1 00:00:04,841 --> 00:00:07,010 GOOD MORNING AND WELCOME TO 2 00:00:07,010 --> 00:00:10,147 THE WORKSHOP CONCEPT TO THE 3 00:00:10,147 --> 00:00:10,647 CLINIC, HOW TO ADVANCE 4 00:00:10,647 --> 00:00:11,181 INNOVATIVE LUNG AND SLEEP 5 00:00:11,181 --> 00:00:11,815 THERAPIES TO PATIENTS WITH HIGH 6 00:00:11,815 --> 00:00:13,750 UNMET NEEDS. 7 00:00:13,750 --> 00:00:16,086 FOR THOSE OF YOU WHO ARE JUST 8 00:00:16,086 --> 00:00:18,655 JOINING US TODAY, MY NAME IS 9 00:00:18,655 --> 00:00:20,424 MARRAH LACHOWICZ-SCROGGINS, I'M 10 00:00:20,424 --> 00:00:22,659 THE BRANCH CHIEF FOR LUNG 11 00:00:22,659 --> 00:00:24,194 DEVELOPMENT AND PAID RICK 12 00:00:24,194 --> 00:00:25,228 DEVELOPMENT BRANCH AS WELL AS 13 00:00:25,228 --> 00:00:27,397 ONE OF THE CO-ORGANIZERS OF THIS 14 00:00:27,397 --> 00:00:27,898 WORKSHOP. 15 00:00:27,898 --> 00:00:30,934 WE ARE LOOKING FORWARD TO AN 16 00:00:30,934 --> 00:00:31,701 EXCITING SECOND DAY TOGETHER. 17 00:00:31,701 --> 00:00:36,106 NEXT SLIDE. 18 00:00:36,106 --> 00:00:37,140 >> GOOD MORNING. 19 00:00:37,140 --> 00:00:38,909 THIS PRESENTATION IS BEING 20 00:00:38,909 --> 00:00:39,242 RECORDED. 21 00:00:39,242 --> 00:00:42,345 PLEASE MAINTAIN RESEARCH 22 00:00:42,345 --> 00:00:44,514 INTEGRITY AND CONFIDENTIALITY. 23 00:00:44,514 --> 00:00:46,783 USE CHAT FOR DISCUSSION WITH 24 00:00:46,783 --> 00:00:49,352 OTHER WORKSHOP PARTICIPANTS. 25 00:00:49,352 --> 00:00:52,155 THIS, TOO, WILL BE USED FOR OPEN 26 00:00:52,155 --> 00:00:54,024 DISCUSSION BETWEEN WORKSHOP 27 00:00:54,024 --> 00:00:54,925 PARTICIPANTS. 28 00:00:54,925 --> 00:00:57,093 COMMENTS WILL BE CAPTURED FOR 29 00:00:57,093 --> 00:00:58,962 REVIEW BY WORKSHOP SPONSORS. 30 00:00:58,962 --> 00:01:01,498 USE Q&A TO DIRECT QUESTIONS TO 31 00:01:01,498 --> 00:01:01,765 SPEAKERS. 32 00:01:01,765 --> 00:01:04,167 THIS TOOL CAN BE USED DURING THE 33 00:01:04,167 --> 00:01:06,036 SESSION TO ASK THE SPEAKERS A 34 00:01:06,036 --> 00:01:06,303 QUESTION. 35 00:01:06,303 --> 00:01:08,572 QUESTION WILL BE CAPTURED FOR 36 00:01:08,572 --> 00:01:10,740 REVIEW BY WORKSHOP SPONSORS. 37 00:01:10,740 --> 00:01:12,742 KINDLY STAY ON MUTE AND OFF 38 00:01:12,742 --> 00:01:12,976 CAMERA. 39 00:01:12,976 --> 00:01:13,243 THANK YOU. 40 00:01:13,243 --> 00:01:15,412 >> NEXT N SLIDE, PLEASE. 41 00:01:15,412 --> 00:01:19,749 I WOULD LIKE TO KICK OFF THIS 42 00:01:19,749 --> 00:01:22,886 MEETING WITH A BRIEF WELCOME OF 43 00:01:22,886 --> 00:01:25,088 DR. MATUTE-BELLO WHO IS THE 44 00:01:25,088 --> 00:01:26,990 ACTING DIRECTOR OF LUNG 45 00:01:26,990 --> 00:01:27,257 DISEASES. 46 00:01:27,257 --> 00:01:32,229 >> THANK MARRAH AND I HOPE 47 00:01:32,229 --> 00:01:33,230 EVERYBODY CAN HEAR ME. 48 00:01:33,230 --> 00:01:34,831 GOOD MORNING TO ALL. 49 00:01:34,831 --> 00:01:37,267 I AM REALLY DELIGHTED TO BE HERE 50 00:01:37,267 --> 00:01:39,336 TODAY WITH YOU ON THE SECOND DAY 51 00:01:39,336 --> 00:01:40,904 OF THIS WORKSHOP. 52 00:01:40,904 --> 00:01:43,573 AS WE GATHER HERE TO EXPLORE 53 00:01:43,573 --> 00:01:45,308 EFFECTIVE APPROACHES TO 54 00:01:45,308 --> 00:01:47,377 PERFORMING TRANSLATIONAL 55 00:01:47,377 --> 00:01:48,278 RESEARCH. 56 00:01:48,278 --> 00:01:51,982 THE DIVISIONECOGNIZES THAT 57 00:01:51,982 --> 00:01:54,384 EFFECTIVE TRANSLATION OF IDEAS 58 00:01:54,384 --> 00:01:57,821 FROM THE BENCH INTO THE CLINIC 59 00:01:57,821 --> 00:01:58,955 REQUIRES COLLABORATION BETWEEN 60 00:01:58,955 --> 00:02:00,857 BASIC SCIENTISTS, CLINICIANS AND 61 00:02:00,857 --> 00:02:02,158 INDUSTRY PARTNERS. 62 00:02:02,158 --> 00:02:06,029 DOING THIS EFFECTIVELY REQUIRES 63 00:02:06,029 --> 00:02:07,230 COORDINATION ACROSS DIFFERENT 64 00:02:07,230 --> 00:02:08,331 DISCIPLINES ANDIP INSTITUTIONS 65 00:02:08,331 --> 00:02:10,834 THIS CAN BE PARTICULARLY 66 00:02:10,834 --> 00:02:11,134 CHALLENGING. 67 00:02:11,134 --> 00:02:12,903 WITH THIS ING.MIND, WE HAVE 68 00:02:12,903 --> 00:02:14,604 GATHERED TODAY AN IMPRESSIVE 69 00:02:14,604 --> 00:02:16,573 GROUP OF EXPERTS WHO WILL BE 70 00:02:16,573 --> 00:02:19,042 PROVIDING US WITH AN OVERVIEW ON 71 00:02:19,042 --> 00:02:22,012 HOW TO NAVIGATE THE PATH TO THE 72 00:02:22,012 --> 00:02:24,781 CLINIC WITH EMPHASIS HOW TO TAKE 73 00:02:24,781 --> 00:02:25,882 CLINICAL DATA DESCRIBING 74 00:02:25,882 --> 00:02:27,851 MECHANISMS OF ACTION, SUCH AS 75 00:02:27,851 --> 00:02:30,520 HOW A DRUG OR DEVICE MAY TARGET 76 00:02:30,520 --> 00:02:36,326 SPECIFIC PATH WAYS OR 77 00:02:36,326 --> 00:02:37,394 PHYSIOLOGICAL PROCESS, CLINICAL 78 00:02:37,394 --> 00:02:38,795 DATA SUPPORTING THE PROPOSED 79 00:02:38,795 --> 00:02:40,196 MECHANISM AND EFFICACY. 80 00:02:40,196 --> 00:02:43,900 WE HOPE THIS WORKSHOP WILL 81 00:02:43,900 --> 00:02:45,936 ENCOURAGE THE TYPE OF 82 00:02:45,936 --> 00:02:46,570 INTERDISCIPLINARY COLLABORATION 83 00:02:46,570 --> 00:02:49,072 THAT IS REQUIRED TO TRANSLATE 84 00:02:49,072 --> 00:02:52,742 MORE DISCOVERIES INTO EFFECTIVE 85 00:02:52,742 --> 00:02:53,843 INTERVENTIONS WHO WILL HELP THE 86 00:02:53,843 --> 00:02:55,378 MANY PATIENTS TODAY ARE 87 00:02:55,378 --> 00:02:58,915 SUFFERING FROM LUNG AND SLEEP 88 00:02:58,915 --> 00:02:59,182 DISORDERS. 89 00:02:59,182 --> 00:03:01,818 TODAY WE WILL BE DISCUSSING HOW 90 00:03:01,818 --> 00:03:04,721 BENCH DISCOVERIES CAN ENTER THE 91 00:03:04,721 --> 00:03:06,423 CLINICAL PIPELINE DEVELOPMENT, 92 00:03:06,423 --> 00:03:09,893 EARLY PHASE TESTING, SAFETY, 93 00:03:09,893 --> 00:03:10,994 PRELIMINARY EFFICACY. 94 00:03:10,994 --> 00:03:13,496 DOING THIS SUCCESSFULLYY REQUIRS 95 00:03:13,496 --> 00:03:17,601 LEVERAGE IN PARTNERSHIPS WITH 96 00:03:17,601 --> 00:03:20,437 ACADEMIA, RESEARCH ORGANIZATIONS 97 00:03:20,437 --> 00:03:23,006 AND INDUSTRY COLLABORATORS WHO 98 00:03:23,006 --> 00:03:25,308 SUPPORT THE DEVELOPMENT OF THE 99 00:03:25,308 --> 00:03:26,142 INVESTAL PRODUCT. 100 00:03:26,142 --> 00:03:27,510 AS DISCUSSED YESTERDAY, THIS 101 00:03:27,510 --> 00:03:29,112 PROCESS INCLUDES FUNDING SOURCES 102 00:03:29,112 --> 00:03:31,615 FOR GRANTS THAT HAVE BEEN 103 00:03:31,615 --> 00:03:33,750 SECURED TO ADVANCE RESEARCH, 104 00:03:33,750 --> 00:03:36,987 WHETHER IT BE FROM INVESTORS OR 105 00:03:36,987 --> 00:03:41,825 UNIQUE RESEARCH PROGRAMS SUCH AS 106 00:03:41,825 --> 00:03:42,993 NHLBI -- PROGRAM OR SMALL 107 00:03:42,993 --> 00:03:43,593 BUSINESS PROGRAM. 108 00:03:43,593 --> 00:03:47,998 THIS IS NFICIENT.HER CRITICAL O 109 00:03:47,998 --> 00:03:49,566 SUCCESSFULLY ADVANCE NOVEL 110 00:03:49,566 --> 00:03:51,067 INTERVENTIONS IS TO ENGAGE 111 00:03:51,067 --> 00:03:52,068 PATIENTS AND HEALTH CARE 112 00:03:52,068 --> 00:03:54,004 PROVIDERS IN THE DEVELOPMENT 113 00:03:54,004 --> 00:03:57,307 PROCESS, INCLUDING PATIENT 114 00:03:57,307 --> 00:03:59,209 ADVOCACY GROUPS AND PROFESSIONAL 115 00:03:59,209 --> 00:03:59,576 SOCIETIES. 116 00:03:59,576 --> 00:04:02,212 YOU WILL HEAR MORE HOW HO THIS 117 00:04:02,212 --> 00:04:04,347 SUCCESSFULLY IN TODAY'S AGENDA. 118 00:04:04,347 --> 00:04:05,949 IMPORTANTLY, WE WANT YOU TO NOT 119 00:04:05,949 --> 00:04:09,619 JUST LISTEN, BUT ACTIVELY 120 00:04:09,619 --> 00:04:11,955 PARTICIPATE IN THE BREAKOUT 121 00:04:11,955 --> 00:04:12,856 SESSIONS ESSENTIAL FOR THE 122 00:04:12,856 --> 00:04:15,025 SUCCESS OF THIS WORKSHOP SO WE 123 00:04:15,025 --> 00:04:19,062 CAN TRULY IDENTIFY BARRIERS TO 124 00:04:19,062 --> 00:04:20,363 TRANSLATING SCIENTIFIC FINDINGS 125 00:04:20,363 --> 00:04:22,298 INTO MEDICAL INTERVENTIONS AND 126 00:04:22,298 --> 00:04:24,234 DISCUSS HOW TO OVERCOME THESE 127 00:04:24,234 --> 00:04:24,868 BARRIERS. 128 00:04:24,868 --> 00:04:29,973 FOR EXAMPLE, WE KNOW LUNG 129 00:04:29,973 --> 00:04:31,441 DISEASES OFTEN INVOLVE COMPLEX 130 00:04:31,441 --> 00:04:33,576 PHYSIOLOGICAL PROCESSES. 131 00:04:33,576 --> 00:04:38,481 HOW CAN WE IDENTIFY IODELS TO 132 00:04:38,481 --> 00:04:40,283 ENSURE PATHWAYS OF TARGETS THAT 133 00:04:40,283 --> 00:04:42,719 MAY TREAT AN ARRAY OF LUNG 134 00:04:42,719 --> 00:04:43,186 DISEASES? 135 00:04:43,186 --> 00:04:45,922 WHAT ARE SOME OPTIMAL TRIAL 136 00:04:45,922 --> 00:04:47,957 DESIGNS THAT CONSIDER PATIENT 137 00:04:47,957 --> 00:04:52,328 HETEROGENERALITY AND 138 00:04:52,328 --> 00:04:52,662 STANDARDIZED. 139 00:04:52,662 --> 00:04:57,534 WHAT NEW TOOLS OR BIOMARKERS ARE 140 00:04:57,534 --> 00:04:59,803 SENSITIVE ENOUGH FOR EFFICACY. 141 00:04:59,803 --> 00:05:03,973 CAN WE ACCEPT NEW CLINICAL TRIAL 142 00:05:03,973 --> 00:05:05,875 OUTCOMES BEYOND FEV1 143 00:05:05,875 --> 00:05:07,410 PARTICULARLY RARE DISEASES ASE 144 00:05:07,410 --> 00:05:11,481 ITH ITUNMET NEEDS THAT 145 00:05:11,481 --> 00:05:13,416 CANNOT DR FOR THE SAMPLE SIZES 146 00:05:13,416 --> 00:05:16,686 TO DETECT MEANINGFUL OUTCOMES. 147 00:05:16,686 --> 00:05:18,755 CHALLENGE THAT IS NOT BEING THE 148 00:05:18,755 --> 00:05:25,195 FOCUS OF THIS WORKSHOP, IT IS 149 00:05:25,195 --> 00:05:27,130 HOW TO IMPLEMENT. 150 00:05:27,130 --> 00:05:29,899 WE WANT TO HEAR BARRIERS OF 151 00:05:29,899 --> 00:05:32,001 IMPLEMENTATION, LACK OF 152 00:05:32,001 --> 00:05:33,903 AWARENESS, THE RESISTANCE FOR 153 00:05:33,903 --> 00:05:36,439 CHANGE AND THE NEED FOR TRAINS 154 00:05:36,439 --> 00:05:38,208 AND RESOURCES AND HOW TO 155 00:05:38,208 --> 00:05:41,878 OVERCOME BARRIERS TO TRULY 156 00:05:41,878 --> 00:05:43,847 EFFECTIVELY END UP REACHING THE 157 00:05:43,847 --> 00:05:45,248 PATIENTS WHO NEED THESE NEW 158 00:05:45,248 --> 00:05:46,082 DISCOVERIES THE MOST. 159 00:05:46,082 --> 00:05:48,485 THAT IS MY CHALLENGE FOR YOU 160 00:05:48,485 --> 00:05:48,985 TODAY. 161 00:05:48,985 --> 00:05:51,054 I CHALLENGE ALL OF YOU TODAY TO 162 00:05:51,054 --> 00:05:53,923 LAY THE GROUNDWORK THAT WILL 163 00:05:53,923 --> 00:05:56,359 HELP US BUILD A PATH TOWARDS 164 00:05:56,359 --> 00:05:59,229 CURING AT LEAST ONE C LUNG 165 00:05:59,229 --> 00:06:01,431 DISEASE HOPEFULLY MORE. 166 00:06:01,431 --> 00:06:03,500 GO FOR IT. 167 00:06:03,500 --> 00:06:04,234 THANK YOU VERY MUCH. 168 00:06:04,234 --> 00:06:05,735 IT HAS BEEN A GREATPLEASURE. 169 00:06:05,735 --> 00:06:07,637 >> THANK YOU, GUS. 170 00:06:07,637 --> 00:06:11,007 I WANT TAKE THIS OPPORTUNITY 171 00:06:11,007 --> 00:06:12,876 TO BRIEFLY REINTRODUCE OUR OTHER 172 00:06:12,876 --> 00:06:15,145 CO-ORGANIZERS FOR THE WORKSHOP. 173 00:06:15,145 --> 00:06:16,412 MY COLLEAGUE DR. JOHN SHERIDAN 174 00:06:16,412 --> 00:06:18,848 IS THE BRANCH CHIEF OF THE 175 00:06:18,848 --> 00:06:20,183 RESTRICTIVE AND VASCULAR 176 00:06:20,183 --> 00:06:21,417 DISEASES BRANCH AND CO-LEADING 177 00:06:21,417 --> 00:06:21,885 THIS MEETING. 178 00:06:21,885 --> 00:06:25,455 NEXT SLIDE. 179 00:06:25,455 --> 00:06:26,856 WE ALSO HAVE TWO WONDERFUL 180 00:06:26,856 --> 00:06:28,625 WORKSHOP CO-CHAIRS THAT YOU WILL 181 00:06:28,625 --> 00:06:30,426 BE HEARING FROM THROUGHOUT THE 182 00:06:30,426 --> 00:06:31,494 MEETING TODAY. 183 00:06:31,494 --> 00:06:34,130 WE HAVE DR. KATELYN KRIVCHENIA, 184 00:06:34,130 --> 00:06:36,366 WHO IS THE DIRECTOR OF THE 185 00:06:36,366 --> 00:06:38,034 INTERSTITIAL AND RARE LUNG 186 00:06:38,034 --> 00:06:39,702 DISEASE PROGRAM AS WELL AS THE 187 00:06:39,702 --> 00:06:42,772 DIRECTOR OF THE INFANT CYSTIC 188 00:06:42,772 --> 00:06:44,574 FIBROSIS NEWBORN SCREENING 189 00:06:44,574 --> 00:06:46,042 PROGRAM AT NATIONWIDE CHILDREN'. 190 00:06:46,042 --> 00:06:48,378 SHE CARES FORNFANTS A 191 00:06:48,378 --> 00:06:52,248 CHS WELL ASOUNG ADULTS 192 00:06:52,248 --> 00:06:54,684 WITH LUNG DISEASES. 193 00:06:54,684 --> 00:06:57,253 JEFT MOFFIT IS THE CE-PRESIDENT 194 00:06:57,253 --> 00:06:59,689 DEVELOPMENT AND CARBON 195 00:06:59,689 --> 00:06:59,989 BIOSCIENCES. 196 00:06:59,989 --> 00:07:03,159 HE HAS OVER 15 YEARSOVER BIOTECH 197 00:07:03,159 --> 00:07:06,196 EXPERIENCE AND A BOARD CERTIFIED 198 00:07:06,196 --> 00:07:06,529 TOXICOLOGIST. 199 00:07:06,529 --> 00:07:08,665 HE WORKED IN ALL PHASES OF THE 200 00:07:08,665 --> 00:07:10,500 DEVELOPMENT PROCESS AS WELL AS 201 00:07:10,500 --> 00:07:13,102 ON SEVERAL THERAPEUTIC AREAS 202 00:07:13,102 --> 00:07:13,903 FOCUSING MAINLY ON RARE 203 00:07:13,903 --> 00:07:14,170 DISEASES. 204 00:07:14,170 --> 00:07:17,774 NEXT SLIDE. 205 00:07:17,774 --> 00:07:20,844 A PURPOSE OF THIS WORKSHOP IS TO 206 00:07:20,844 --> 00:07:23,680 HELP EDUCATE THE PULMONARY AND 207 00:07:23,680 --> 00:07:25,048 SLEEP MEDICINE RESEARCH 208 00:07:25,048 --> 00:07:26,216 COMMUNITY HOW WE CAN BRING IDEAS 209 00:07:26,216 --> 00:07:28,585 FROM THE BENCH TO THE CLINIC. 210 00:07:28,585 --> 00:07:30,420 WE WANT TO DISCOVER THE BARRIERS 211 00:07:30,420 --> 00:07:37,827 AND FACILITATORS INNOVATION 212 00:07:37,827 --> 00:07:38,494 FOR DEVELOPING. 213 00:07:38,494 --> 00:07:40,897 THERE IS MISALIGNMENT WITH 214 00:07:40,897 --> 00:07:42,866 DISEASE BURDEN AND NOVEL THERAPY 215 00:07:42,866 --> 00:07:44,601 INNOVATION WHERE IDEAS MAY BE 216 00:07:44,601 --> 00:07:46,236 IMPEDED BY A VARIETY OF 217 00:07:46,236 --> 00:07:47,837 DIFFERENT FACTORS SUCH AS 218 00:07:47,837 --> 00:07:49,372 DECISION MAKING FOR A CLINICAL 219 00:07:49,372 --> 00:07:51,941 MODEL ALL THE WAY TO SECURING 220 00:07:51,941 --> 00:07:53,643 CAPITAL TO SUPPORT DRUG 221 00:07:53,643 --> 00:07:56,312 DEVELOPMENT IN ADVANCE OF AN 222 00:07:56,312 --> 00:07:56,512 IND. 223 00:07:56,512 --> 00:07:58,348 THE SLOW SPEED WE ARE MAKING 224 00:07:58,348 --> 00:07:59,716 THESE NEW THERAPIES COME TO 225 00:07:59,716 --> 00:08:01,818 MARKET CAN CONTRIBUTE TO 226 00:08:01,818 --> 00:08:03,386 INCREASED MORBIDITY AND 227 00:08:03,386 --> 00:08:05,221 MORTALITY AND EXACERB 228 00:08:05,221 --> 00:08:05,688 EXISTING HEALTH 229 00:08:05,688 --> 00:08:08,791 DISPARITIES. REALLY WANT T TRY 230 00:08:08,791 --> 00:08:11,461 THESE EXISTING GAPS BETWEEN 231 00:08:11,461 --> 00:08:11,928 CLINICAL THERAPY 232 00:08:11,928 --> 00:08:13,162 INNOVATION AND 233 00:08:13,162 --> 00:08:17,634 UNMET HEALTH NEEDS IN LUNG AND 234 00:08:17,634 --> 00:08:19,702 SLEEP MEDICINE AS DR. 235 00:08:19,702 --> 00:08:21,004 MATUTE-BELLO SAID TO REDUCE 236 00:08:21,004 --> 00:08:23,006 HEALTH DISPARITIES, IMPROVE 237 00:08:23,006 --> 00:08:24,674 HEALTH OUTCOMES AND LOWER 238 00:08:24,674 --> 00:08:25,541 AL COSTS. 239 00:08:25,541 --> 00:08:28,144 SOME OF THE SPECIFIC BARRIERS WE 240 00:08:28,144 --> 00:08:29,545 ALREADYOGNIZEALENGES 241 00:08:29,545 --> 00:08:31,347 THAT WE HOPE THE PARTICIPANTS 242 00:08:31,347 --> 00:08:33,583 CAN WEIGH IN DURING THE 243 00:08:33,583 --> 00:08:36,052 BREAKOUTS LATER TODAY INCLUDE 244 00:08:36,052 --> 00:08:37,120 UNDERSTANDING WHAT ARE THE 245 00:08:37,120 --> 00:08:39,522 GREATEST CHALLENGES TO OVERCOME 246 00:08:39,522 --> 00:08:41,291 IN DIFFERENT STAGES OF 247 00:08:41,291 --> 00:08:42,091 DEVELOPMENT FOR NEGLECTED 248 00:08:42,091 --> 00:08:43,927 DISEASE AREAS. 249 00:08:43,927 --> 00:08:45,728 AS DR. MATUTE-BELLO MENTIONED 250 00:08:45,728 --> 00:08:47,730 THIS COULD BE FROM BASIC TO 251 00:08:47,730 --> 00:08:48,665 TRANSLATIONAL SCIENCE TO 252 00:08:48,665 --> 00:08:51,434 CLINICAL TRIALS OR EVEN 253 00:08:51,434 --> 00:08:52,001 IMPLEMENTATION SCIENCE. 254 00:08:52,001 --> 00:08:54,504 ALSO CAN WE PARTNER WITH 255 00:08:54,504 --> 00:08:55,705 REGULATORS FOR INNOVATION IN 256 00:08:55,705 --> 00:08:57,607 RARE DISEASE AREAS TO HAVE 257 00:08:57,607 --> 00:09:00,176 FASTER CLINICAL TRIALS IN ORDER 258 00:09:00,176 --> 00:09:01,377 TO DETERMINE EFFECTIVENESS OF 259 00:09:01,377 --> 00:09:03,646 NEW THERAPIES? 260 00:09:03,646 --> 00:09:06,516 ALSO HOW IS PUBLIC INVESTMENT IN 261 00:09:06,516 --> 00:09:07,817 BIOMARKER RESEARCH BEST 262 00:09:07,817 --> 00:09:09,619 LEVERAGED IN THE DEVELOPMENT OF 263 00:09:09,619 --> 00:09:09,986 NOVEL THERAPIES? 264 00:09:09,986 --> 00:09:13,356 AND FINALLY, HOW CAN WE ASSURE 265 00:09:13,356 --> 00:09:15,158 EQUITABLE PATIENT ACCESS TO T 266 00:09:15,158 --> 00:09:17,327 THESE INNOVATIVE THERAPIES THAT 267 00:09:17,327 --> 00:09:18,261 ADDRESS UNMET NEEDS. 268 00:09:18,261 --> 00:09:20,029 NEXT SLIDE. 269 00:09:20,029 --> 00:09:23,700 SO AS I MENTIONED YESTERDAY, TO 270 00:09:23,700 --> 00:09:25,001 FACILITATE THERAPEUTIC 271 00:09:25,001 --> 00:09:27,303 INNOVATION WE ARE HOPING TO 272 00:09:27,303 --> 00:09:29,639 DEMYSTIFY THE PROCESS OF GETTING 273 00:09:29,639 --> 00:09:30,473 YOUR IDEAS FROM THE BENCH 274 00:09:30,473 --> 00:09:32,108 THROUGH THE REGULATORY AND 275 00:09:32,108 --> 00:09:33,076 COMMERCIALIZATION PIPELINES. 276 00:09:33,076 --> 00:09:35,812 THE FOCUS OF THIS WORKSHOP HAS 277 00:09:35,812 --> 00:09:38,881 BEEN PRIMARILY IND ENABLING 278 00:09:38,881 --> 00:09:42,151 PATHWAYS, THE PATH WOULD 279 00:09:42,151 --> 00:09:46,255 TAKE YOUR IDEA THROUGH RELEVANT 280 00:09:46,255 --> 00:09:49,425 SYSTEMS, PK STUDIES AND 281 00:09:49,425 --> 00:09:52,428 AC CAPITAL TO MOVE INTO 282 00:09:52,428 --> 00:09:54,831 MANUFACTURING SO YOU ARE READY 283 00:09:54,831 --> 00:09:58,668 TO MOVE INTO TRIALS. 284 00:09:58,668 --> 00:09:59,902 AS DR. MATUTE-BELLO MENTIONED 285 00:09:59,902 --> 00:10:03,306 YOU NEED TO PARTNER WITH 286 00:10:03,306 --> 00:10:04,507 PATIENTS AS WELL AS OTHER PEERS 287 00:10:04,507 --> 00:10:06,109 SUCCESSFUL IN THIS PROCESS. 288 00:10:06,109 --> 00:10:07,744 YOU'LL SEE THESE WORKSHOPS 289 00:10:07,744 --> 00:10:08,778 CONTINUE TODAY THROUGH THE 290 00:10:08,778 --> 00:10:11,447 WORKSHOP SESSIONS. 291 00:10:11,447 --> 00:10:13,916 NEXT SLIDE. 292 00:10:13,916 --> 00:10:15,818 SO THE SPEAKERS ON TODAY'S 293 00:10:15,818 --> 00:10:17,820 AGENDA WILL BE REALLY FOCUSING 294 00:10:17,820 --> 00:10:20,189 ON HOW TO CONDUCT CLINICAL 295 00:10:20,189 --> 00:10:21,858 TRIALS FOR RARE DISEASES AND HOW 296 00:10:21,858 --> 00:10:25,328 THAT PRESENTS A UNIQUE SET OF 297 00:10:25,328 --> 00:10:26,496 CHALLENGES THAT COMPLICATE THE 298 00:10:26,496 --> 00:10:28,297 PATH TO INNOVATION. 299 00:10:28,297 --> 00:10:29,932 FIRSTLY, WE KNOW THERE ARE SMALL 300 00:10:29,932 --> 00:10:32,268 PATIENT POPULATIONS IN SOME OF 301 00:10:32,268 --> 00:10:34,904 THESE RARE DISEASES MAKING IT 302 00:10:34,904 --> 00:10:37,073 DIFFICULT TO RECRUIT ENOUGH 303 00:10:37,073 --> 00:10:38,274 PARTICIPANTS TO ACHIEVE 304 00:10:38,274 --> 00:10:39,409 STATISTICALLY SIGNIFICANT 305 00:10:39,409 --> 00:10:41,110 RESULTS.TEN THIS MAY LEAD TOD T 306 00:10:41,110 --> 00:10:44,781 WITH LIMITED POWER AND INCREASED 307 00:10:44,781 --> 00:10:45,281 VARIABILITY. 308 00:10:45,281 --> 00:10:49,752 WE KNOW THERE IS A HETEROGENEITY 309 00:10:49,752 --> 00:10:52,688 WHICH CAN FURTHER COMPLICATE 310 00:10:52,688 --> 00:10:53,523 PATIENT SELECTION AND RESULTS 311 00:10:53,523 --> 00:10:54,590 AND INTERPRETATIONS. 312 00:10:54,590 --> 00:10:56,926 THE LACK OF ESTABLISHED END 313 00:10:56,926 --> 00:10:59,228 POINTS AND ESTABLISHED OUTCOME 314 00:10:59,228 --> 00:11:01,364 MEASURES POSES ANOTHER HURDLE 315 00:11:01,364 --> 00:11:02,932 OFTEN NECESSITATING NOVEL 316 00:11:02,932 --> 00:11:04,767 ASSESSMENT TOOLS AND BIOMARKERS 317 00:11:04,767 --> 00:11:07,503 WHICH WE KNOW CAN BE TIME 318 00:11:07,503 --> 00:11:08,671 CONSUMING AND RESOURCE 319 00:11:08,671 --> 00:11:09,338 INTENSETIVE. 320 00:11:09,338 --> 00:11:10,807 REGULATORY REQUIREMENTS THOUGH 321 00:11:10,807 --> 00:11:12,275 ARE BECOMING INCREASINGLY 322 00:11:12,275 --> 00:11:14,377 ACCOMMODATING FOR RARE DISEASES. 323 00:11:14,377 --> 00:11:15,912 STILL PRESENT SIGNIFICANT 324 00:11:15,912 --> 00:11:18,014 CHALLENGES IF THEY MUST STRIKE A 325 00:11:18,014 --> 00:11:20,183 BALANCE BETWEEN EXPEDITED ACCESS 326 00:11:20,183 --> 00:11:22,218 TO POTENTIALLY LIFE SAVING 327 00:11:22,218 --> 00:11:24,220 TREATMENTS ALL THE WHILE STILL 328 00:11:24,220 --> 00:11:26,689 ASSURING SAFETY AND EFFICACY. 329 00:11:26,689 --> 00:11:28,124 FINANCIAL CONSTRAINTS ALSO PLAY 330 00:11:28,124 --> 00:11:32,128 A CRITICAL ROLE AS THE HIGH COST 331 00:11:32,128 --> 00:11:34,931 ASSOCIATED WITH DEVELOPING THESE 332 00:11:34,931 --> 00:11:35,865 THERAPIES FOR SMALL PATIENT 333 00:11:35,865 --> 00:11:37,266 GROUPS LEADS TO LIMITED 334 00:11:37,266 --> 00:11:38,801 INVESTMENT FROM POTENTIAL 335 00:11:38,801 --> 00:11:39,802 PARTNERS. 336 00:11:39,802 --> 00:11:41,771 COLLECTIVELY THESE CHALLENGES 337 00:11:41,771 --> 00:11:43,139 NECESSITATE COLLABORATIVE 338 00:11:43,139 --> 00:11:44,540 EFFORTS AMONG STAKEHOLDERS 339 00:11:44,540 --> 00:11:46,409 INCLUDING PATIENTS, RESEARCHERS, 340 00:11:46,409 --> 00:11:48,845 REGULATORY BODIES, CLINICIANS 341 00:11:48,845 --> 00:11:50,947 AND INDUSTRY TO FOSTER 342 00:11:50,947 --> 00:11:52,582 INNOVATION AND ULTIMATELY 343 00:11:52,582 --> 00:11:53,449 DELIVER EFFECTIVE THERAPIES TO 344 00:11:53,449 --> 00:11:54,617 THOSE IN NEED. 345 00:11:54,617 --> 00:11:56,252 AS DR. MATUTE-BELLO MENTIONED 346 00:11:56,252 --> 00:11:58,654 EARLIER, WE DO REALLY WANT 347 00:11:58,654 --> 00:12:01,824 PARTICIPANTS IN THIS MEETING TO 348 00:12:01,824 --> 00:12:03,126 BE ACTIVE. 349 00:12:03,126 --> 00:12:04,527 WE WILL HAVE THE CHAT OPEN FOR 350 00:12:04,527 --> 00:12:05,962 YOU TO CONNECT TO OTHERS 351 00:12:05,962 --> 00:12:07,663 THROUGHOUT THE WORKSHOP. 352 00:12:07,663 --> 00:12:09,432 ADLY, WE WILL HAVE THE 353 00:12:09,432 --> 00:12:11,734 ZOOM ROOM OPEN TODAY FOR ANOTHER 354 00:12:11,734 --> 00:12:13,970 NETWORKING BREAK AND FINALLY WE 355 00:12:13,970 --> 00:12:15,671 HOPE THAT YOU WILL PARTICIPATE 356 00:12:15,671 --> 00:12:16,906 IN THE BREAKOUT SESSIONS AT THE 357 00:12:16,906 --> 00:12:18,207 END OF THE DAY. 358 00:12:18,207 --> 00:12:19,542 THIS WILL REALLY HELP DETERMINE 359 00:12:19,542 --> 00:12:23,346 THE BARRIERS AND FACILITATORS TO 360 00:12:23,346 --> 00:12:25,681 TRANSLATE SCIENTIFIC FINDINGS 361 00:12:25,681 --> 00:12:31,587 FOR SLEEP MEDICINE AND CIRCADIAN 362 00:12:31,587 --> 00:12:33,022 BIOLOGY TO PATIENTS WHO NEED 363 00:12:33,022 --> 00:12:34,090 THEM MOST. 364 00:12:34,090 --> 00:12:39,128 I WOULD LIKE TO TURN THIS 365 00:12:39,128 --> 00:12:40,796 G OVEO DR. KRIVCHENIA 366 00:12:40,796 --> 00:12:42,632 WITH HER TALK PATH TO THE CLINIC 367 00:12:42,632 --> 00:12:43,299 ROAD MAP. 368 00:12:43,299 --> 00:12:45,001 THANK YOU. 369 00:12:45,001 --> 00:12:47,703 >> THANK YOU, MARRAH AND GOOD 370 00:12:47,703 --> 00:12:50,139 MORNING,AGAIN, TO EVERYONE. 371 00:12:50,139 --> 00:12:52,808 THANK YOU FOR JOINING US TODAY. 372 00:12:52,808 --> 00:12:55,011 I APPRECIATE THE INTRODUCTION. 373 00:12:55,011 --> 00:12:56,512 SO WE'RE GOING TO TALK ABOUT 374 00:12:56,512 --> 00:12:59,549 KIND OF THE OTHER HALF OF THIS 375 00:12:59,549 --> 00:12:59,849 PROCESS. 376 00:12:59,849 --> 00:13:01,384 NEXT SLIDE. 377 00:13:01,384 --> 00:13:03,819 SO WE'VE SEEN A VERSION OF THIS 378 00:13:03,819 --> 00:13:05,488 SLIDE BEFORE YESTERDAY AND 379 00:13:05,488 --> 00:13:08,424 TALKED A LOTBOUT THAT A PRE-IND 380 00:13:08,424 --> 00:13:10,826 PROCESS AND THE GOAL OF THIS 381 00:13:10,826 --> 00:13:12,195 WORKSHOP BEING REALLY HOW TO 382 00:13:12,195 --> 00:13:12,662 HELP 383 00:13:12,662 --> 00:13:14,297 FACILITATE THE ENTIRE 384 00:13:14,297 --> 00:13:17,633 SPECTRUM AS WE WORK THROUGH 385 00:13:17,633 --> 00:13:18,267 DIRECT DEVELOPMENT. 386 00:13:18,267 --> 00:13:20,036 TODAY IS MORE ABOUT THAT 387 00:13:20,036 --> 00:13:21,070 CLINICAL ASSESSMENT OF DIRECT 388 00:13:21,070 --> 00:13:21,370 DEVELOPMENT. 389 00:13:21,370 --> 00:13:23,539 THIS IS THE PART THAT MOST 390 00:13:23,539 --> 00:13:26,209 CLINICIANS HAVE AT LEAST SOME 391 00:13:26,209 --> 00:13:28,211 AWARENESS OF, BUT MAY 392 00:13:28,211 --> 00:13:30,413 UNDERAPPRECIATE THE COMPLEXITIES 393 00:13:30,413 --> 00:13:32,782 OF THAT PATH FROM IND TO NDA. 394 00:13:32,782 --> 00:13:35,351 IT IS LONG, IT IS COMPLICATED, 395 00:13:35,351 --> 00:13:36,719 PARTICULARLY IN THE RARE LUNG 396 00:13:36,719 --> 00:13:37,053 DISEASE SPACE. 397 00:13:37,053 --> 00:13:38,754 NEXT SLIDE. 398 00:13:38,754 --> 00:13:42,892 SO AS WAS SAID, I WORK AS A 399 00:13:42,892 --> 00:13:43,392 PEDIATRIC PULMONOLOGIST. 400 00:13:43,392 --> 00:13:45,194 I TAKE CARE OF KIDS WITH ALL 401 00:13:45,194 --> 00:13:46,295 DISEASE. LUN 402 00:13:46,295 --> 00:13:49,398 MY AREA OF FOCUS ARE INFANTS 403 00:13:49,398 --> 00:13:52,134 WITH CF AND PATIENTS WITH 404 00:13:52,134 --> 00:13:53,569 INTERSTITIAL LUNG DISEASE OR 405 00:13:53,569 --> 00:13:55,571 RARE WEIRD KIND OF LUNG DISEASE. 406 00:13:55,571 --> 00:13:57,340 ANYBODY WHO DOESN'T QUITE HAVE A 407 00:13:57,340 --> 00:13:58,708 SURE ANSWER MAY END UP COMING 408 00:13:58,708 --> 00:13:59,909 THROUGH MY CLINIC. 409 00:13:59,909 --> 00:14:02,178 AND FOR THOSE OF US INSHE ROOM 410 00:14:02,178 --> 00:14:04,280 THAT CARE FOR CHILDREN WITHLD LG 411 00:14:04,280 --> 00:14:04,981 DISEASE AND RARE LUNG DISEASE 412 00:14:04,981 --> 00:14:06,515 UNDERSTAND THERE IS A COMPLETE 413 00:14:06,515 --> 00:14:09,051 LACK OF FDA APPROVED THERAPIES 414 00:14:09,051 --> 00:14:10,319 IN THIS SPACE. 415 00:14:10,319 --> 00:14:12,221 REALLY HIGHLIGHTING THE NEED FOR 416 00:14:12,221 --> 00:14:12,688 WORKSHOPS LIKE THIS. 417 00:14:12,688 --> 00:14:17,159 NEXT SLIDE. 418 00:14:17,159 --> 00:14:18,728 I ALSO AM FORTUNATE TO VOLUNTEER 419 00:14:18,728 --> 00:14:20,896 AS A MEMBER OF THE BOARD OF 420 00:14:20,896 --> 00:14:26,202 DIRECTORS FOR THE CHILDREN'S 421 00:14:26,202 --> 00:14:27,670 INTERSTITIAL AND DIFFUSE LUNG 422 00:14:27,670 --> 00:14:27,903 DISEASE. 423 00:14:27,903 --> 00:14:29,472 THIS WAS STARTED BY A SMALL 424 00:14:29,472 --> 00:14:32,008 GROUP OF PARENTS AND CLINICIAN 425 00:14:32,008 --> 00:14:34,310 RESEARCHERS DEDICATED TO 426 00:14:34,310 --> 00:14:35,745 IMPROVING CARE AND EXPANDING 427 00:14:35,745 --> 00:14:37,813 RESEARCH FOR CHILDREN WITH 428 00:14:37,813 --> 00:14:38,914 INTERSTITIAL LUNG DISEASE. 429 00:14:38,914 --> 00:14:40,883 IT HAS BEEN FASCINATING TO 430 00:14:40,883 --> 00:14:42,251 UNDERSTAND AND LEARN MORE ABOUT 431 00:14:42,251 --> 00:14:44,787 THIS ASPECT OF MEDICINE SO THE 432 00:14:44,787 --> 00:14:46,989 ADVOCACY AND SUPPORT NEEDED TO 433 00:14:46,989 --> 00:14:47,990 COMPLEMENT THE MANAGEMENT IF WE 434 00:14:47,990 --> 00:14:49,892 WANT TO DO THIS WELL. 435 00:14:49,892 --> 00:14:51,727 TODAY YOU WILL HEAR SEVERAL 436 00:14:51,727 --> 00:14:54,363 EXAMPLES OF HOW THAT KIND OF 437 00:14:54,363 --> 00:14:56,565 ADVOCACY AND SUPPORT REALLY CAN 438 00:14:56,565 --> 00:14:57,533 HELP WITH THE RESEARCH 439 00:14:57,533 --> 00:14:58,134 MANAGEMENT OF THIS PROCESS AS 440 00:14:58,134 --> 00:15:01,437 WELL. 441 00:15:01,437 --> 00:15:03,439 NEXT SLIDE. 442 00:15:03,439 --> 00:15:04,774 SO A ALL THE SCIENCE WE 443 00:15:04,774 --> 00:15:08,144 HEARDAR ABOUT YESTERDAY, WHICH S 444 00:15:08,144 --> 00:15:10,513 OOL INAT T 445 00:15:10,513 --> 00:15:13,182 PROJECTS THAT WE'LLHA HEAR 446 00:15:13,182 --> 00:15:14,383 TODAY I WANT TO MAKE SURE WE 447 00:15:14,383 --> 00:15:16,319 HAVE A FACE IN MIND AS WE THINK 448 00:15:16,319 --> 00:15:18,187 ABOUT THE CHILDREN AND PATIENTS 449 00:15:18,187 --> 00:15:19,555 WE ARE HOPING TO HELP. 450 00:15:19,555 --> 00:15:21,424 THIS IS A PICTURE OF M. 451 00:15:21,424 --> 00:15:23,292 SHE AND HER FAMILY GAVE ME 452 00:15:23,292 --> 00:15:24,694 PERMISSION TO SHARE PICTURES AND 453 00:15:24,694 --> 00:15:29,265 HER STORY WITH YOU TO, AGAIN, 454 00:15:29,265 --> 00:15:32,435 HELP HER R THIS PROCESS. 455 00:15:32,435 --> 00:15:34,570 AFTER BIRTH HER LUNGS WERE 456 00:15:34,570 --> 00:15:39,742 FAILING TO OXYGENATE AND 457 00:15:39,742 --> 00:15:40,810 VENTILATE AND SHE NEEDED HELP 458 00:15:40,810 --> 00:15:44,880 WITH A VENTILATOR. 459 00:15:44,880 --> 00:15:49,985 GENETIC TESTING SHOWS TWO -- 460 00:15:49,985 --> 00:15:54,557 SHOWING SURFACTANT PROTEIN B 461 00:15:54,557 --> 00:15:55,524 DEFICIENCY. 462 00:15:55,524 --> 00:15:58,194 THIS IS INCREDIBLY RARE DISEASE. 463 00:15:58,194 --> 00:16:01,430 MUTATIONS IN THIS DISEASESE REST 464 00:16:01,430 --> 00:16:05,801 IN DYSFUNCTIONAL SURFACTANT. 465 00:16:05,801 --> 00:16:10,005 YOU CAN SEE THESE DISORGANIZED 466 00:16:10,005 --> 00:16:12,108 BODIES COMPARED TO NORMAL. 467 00:16:12,108 --> 00:16:17,513 THIS LEADS TO BUILD UP OF 468 00:16:17,513 --> 00:16:23,018 MATERIAL IN THE ALVEOLI SPACES. 469 00:16:23,018 --> 00:16:24,787 THERE IS NO WONDER THIS GIRL 470 00:16:24,787 --> 00:16:26,355 COULDN'T BREATHE LOOKING AT THIS 471 00:16:26,355 --> 00:16:27,556 PICTURE ON THE RIGHT. 472 00:16:27,556 --> 00:16:28,958 SO THERE IS NO PROVEN TRE 473 00:16:28,958 --> 00:16:30,092 N THIS ZZ. 474 00:16:30,092 --> 00:16:32,528 THERE MAY BE SOME BENEFIT FROM 475 00:16:32,528 --> 00:16:36,298 HIGH-DOSE STEROIDS AND OTHER 476 00:16:36,298 --> 00:16:38,334 ANTI-INFLAMMATORY MEDICINES, BUT 477 00:16:38,334 --> 00:16:40,736 NONE OF THIS IS PROVEN AS NO 478 00:16:40,736 --> 00:16:42,705 RANDOMIZED CONTROL TRIALS CAN 479 00:16:42,705 --> 00:16:44,874 REALLY BE DONE IN THIS GROUP. 480 00:16:44,874 --> 00:16:48,277 SO AS CLINICIANS WE ARE LEFT TO 481 00:16:48,277 --> 00:16:50,946 OFFER THERAPIESPI THAT MIGHT WO 482 00:16:50,946 --> 00:16:53,716 AND COUNSEL FAMILIES THAT 483 00:16:53,716 --> 00:16:54,784 PROGNOSIS IS POOR. 484 00:16:54,784 --> 00:16:56,986 M'S LIFE HAS BEEN FULL OF 485 00:16:56,986 --> 00:16:58,888 HOSPITALIZATIONS AND CHRONIC 486 00:16:58,888 --> 00:16:59,121 ILLNESS. 487 00:16:59,121 --> 00:17:00,556 SHE HAS ALWAYS BEEN CUTE AS YOU 488 00:17:00,556 --> 00:17:03,092 SEE HERE, BUT LIFE IS NOT EASY. 489 00:17:03,092 --> 00:17:04,126 SHE IS NOW 16. 490 00:17:04,126 --> 00:17:05,861 I HAVE BEEN CARING FOR HER OVER 491 00:17:05,861 --> 00:17:07,263 THE PAST FIVE YEARS. 492 00:17:07,263 --> 00:17:09,165 SHE SPENT MOST OF HER TIME IN 493 00:17:09,165 --> 00:17:12,535 HERIVING ROOM UNABLE TO WALK 494 00:17:12,535 --> 00:17:14,737 ACROSS THEITHOUT 8 TO 10 495 00:17:14,737 --> 00:17:20,443 LITERS SINS OF OXYGEN. 496 00:17:20,443 --> 00:17:22,745 SHE IS THE LUCKY ONE. 497 00:17:22,745 --> 00:17:26,015 MOST CHILDREN DON'T LIVE A YEAR 498 00:17:26,015 --> 00:17:28,451 WITHOUT LUNG TRANSPLANTATION. 499 00:17:28,451 --> 00:17:32,121 HERE ARE A SCANS, THEEGREE OF 500 00:17:32,121 --> 00:17:34,056 DISTORTION AND ORABNORMALITIES E 501 00:17:34,056 --> 00:17:35,224 HAS AS A RESULT OF HER DISEASE. 502 00:17:35,224 --> 00:17:37,626 NEXT SLIDE. 503 00:17:37,626 --> 00:17:39,628 THIS IS HER A COUPLE OF WEEKS 504 00:17:39,628 --> 00:17:39,795 AGO. 505 00:17:39,795 --> 00:17:42,298 SHE IS NOW SIX WEEKS OUT F 506 00:17:42,298 --> 00:17:43,399 HER OWN LUNG TRANSPLANT. 507 00:17:43,399 --> 00:17:45,167 SO THE PICTURE ON THE RIGHT 508 00:17:45,167 --> 00:17:46,602 SHOWS FOR THE FIRST TIME IN HER 509 00:17:46,602 --> 00:17:49,238 LIFE SHE IS ABLE TO WALK WITHOUT 510 00:17:49,238 --> 00:17:49,472 OXYGEN. 511 00:17:49,472 --> 00:17:53,809 AND NOW SHE DOESN'T HAVE A TRACH 512 00:17:53,809 --> 00:17:54,043 ANYMORE. 513 00:17:54,043 --> 00:17:57,913 AS IS THE CASE WITH ORGAN 514 00:17:57,913 --> 00:17:59,882 TRANSPLANT WE HAVE TRADED ONE 515 00:17:59,882 --> 00:18:05,888 DISEASE FOROR LIFE OFIMMUNOSUPP 516 00:18:05,888 --> 00:18:07,690 REJECTION OF THAT LUNG. 517 00:18:07,690 --> 00:18:10,359 SHE IS THE LUCKY ONE, AGAIN, 518 00:18:10,359 --> 00:18:12,194 THIS IS THE GOOD OUTCOME. 519 00:18:12,194 --> 00:18:13,362 THERE IS GOOD RESEARCH BEING 520 00:18:13,362 --> 00:18:14,930 DONE THERE THE PRECLINICAL 521 00:18:14,930 --> 00:18:15,231 SPACE. 522 00:18:15,231 --> 00:18:17,500 LABS ARE EXPLORING TREATMENTS 523 00:18:17,500 --> 00:18:19,969 THAT FACILITATE PROTEIN FOLDING 524 00:18:19,969 --> 00:18:22,471 AND TRAFFICKING, SIMILAR TO 525 00:18:22,471 --> 00:18:25,808 MEDICATIONS FOR CF T R VARIANTS. 526 00:18:25,808 --> 00:18:29,111 THOSE LOOKING AT GENOME EDITING 527 00:18:29,111 --> 00:18:30,179 OR THERAPY. 528 00:18:30,179 --> 00:18:32,715 HOTH DOHO GET THESE THERAPIES TO 529 00:18:32,715 --> 00:18:34,583 INFANTS AT THIS STAGE WHEN THEY 530 00:18:34,583 --> 00:18:35,851 NEED THEM FOR SURVIVAL. 531 00:18:35,851 --> 00:18:37,386 IF MOST DIE BEFORE A YEAR OF 532 00:18:37,386 --> 00:18:39,054 AGE, HOW DO WE GET THESE DRUGS 533 00:18:39,054 --> 00:18:39,622 TO THEM? 534 00:18:39,622 --> 00:18:42,958 THIS IS TRULY AN UNMET NEED N I 535 00:18:42,958 --> 00:18:43,559 THATTRIC SPACE. 536 00:18:43,559 --> 00:18:45,661 NEXT SLIDE. 537 00:18:45,661 --> 00:18:47,396 SO AS WAS DISCUSSED YESTERDAY, 538 00:18:47,396 --> 00:18:49,365 THIS PROCESS IS INTENSE. 539 00:18:49,365 --> 00:18:50,833 THIS IS ANOTHER WAY W OF 540 00:18:50,833 --> 00:18:52,501 VISUALIZING THIS IN TERMS OF 541 00:18:52,501 --> 00:18:54,336 MERE VOLUMES OF DRUGS THAT MAKE 542 00:18:54,336 --> 00:18:56,272 IT THROUGH EACH STEP OF THE 543 00:18:56,272 --> 00:18:56,505 PROCESS. 544 00:18:56,505 --> 00:18:59,475 OF THE THOUSANDS OF TREATMENTS 545 00:18:59,475 --> 00:19:01,911 ENTERING PRECLINICAL TESTING 546 00:19:01,911 --> 00:19:04,380 ONLY HANDFULS MOVE INTO CLINICAL 547 00:19:04,380 --> 00:19:08,017 RESEARCH STAGE. 548 00:19:08,017 --> 00:19:11,053 ONLY ABOUT 14% OF DRUGS ENTERING 549 00:19:11,053 --> 00:19:12,922 CLINICAL TRIALS WILL END UP 550 00:19:12,922 --> 00:19:13,989 BEING APPROVED. 551 00:19:13,989 --> 00:19:16,492 THIS PROCESS IS LONG, IT IS 552 00:19:16,492 --> 00:19:19,428 EXPENSIVE ESTIMATED COSTS 553 00:19:19,428 --> 00:19:22,598 RANGING FROM $314 MILLION TO 554 00:19:22,598 --> 00:19:23,465 $2.8 BILLION FOR DRUG 555 00:19:23,465 --> 00:19:26,168 DEVELOPMENT, THAT IS A SINGLE 556 00:19:26,168 --> 00:19:26,602 DRUG. 557 00:19:26,602 --> 00:19:27,770 ANYTHING THAT CAN HELP SMOOTH 558 00:19:27,770 --> 00:19:30,105 THAT PROCESS SHOULD BE UTILIZED. 559 00:19:30,105 --> 00:19:33,175 WE TALKED ABOUT A LOT OF THOSE 560 00:19:33,175 --> 00:19:36,512 YESTERDAY, RESEARCHERS ARE BEING 561 00:19:36,512 --> 00:19:38,447 SAVVY USING RESOURCESNGVAILABLE 562 00:19:38,447 --> 00:19:40,082 TO TRY TO MOVE THINGS FASTER. 563 00:19:40,082 --> 00:19:40,783 NEXT SLIDE. 564 00:19:40,783 --> 00:19:43,385 TODAY WE ARE GOING TO A LOT 565 00:19:43,385 --> 00:19:44,887 ABOUT HOW PATIENT FOUNDATIONS 566 00:19:44,887 --> 00:19:46,522 AND INTERACTION WITH PATIENTS 567 00:19:46,522 --> 00:19:48,424 MAY HELP MOVE THIS ALONG AS 568 00:19:48,424 --> 00:19:48,624 WELL. 569 00:19:48,624 --> 00:19:50,559 THERE ARE SEVERAL STEPS ALONG 570 00:19:50,559 --> 00:19:51,994 THE WAY WHERE PATIENT 571 00:19:51,994 --> 00:19:53,295 FOUNDATIONS AND ORGANIZATIONS 572 00:19:53,295 --> 00:19:55,731 MAY BE HELPFUL. 573 00:19:55,731 --> 00:19:57,232 CONNECTING RESEARCHERS WITH 574 00:19:57,232 --> 00:19:59,768 CLINICIANS AND PATIENTS. 575 00:19:59,768 --> 00:20:02,605 IN ULTRA RARE DISEASES PATIENTS 576 00:20:02,605 --> 00:20:04,139 ARE FEW AND FAR BETWEEN. 577 00:20:04,139 --> 00:20:05,040 THIS CONNECTION IS IMPORTANT 578 00:20:05,040 --> 00:20:07,910 WHEN IT COMES TO STUDY DESIGN 579 00:20:07,910 --> 00:20:09,345 RECRUITMENT RETENTION TO MAKE 580 00:20:09,345 --> 00:20:10,679 THE CLINICAL RESEARCH PART OF 581 00:20:10,679 --> 00:20:11,847 THINGS MORE SUCCESSFUL. 582 00:20:11,847 --> 00:20:13,349 WHAT OUTCOME MEASURES ARE 583 00:20:13,349 --> 00:20:15,150 ACTUALLY MEANINGFUL TO THEL 584 00:20:15,150 --> 00:20:18,621 PATIENTS ANDPA FAMILIES?ESTUDY 585 00:20:18,621 --> 00:20:20,222 PROTOCOLS ARE REASONABLE FOR 586 00:20:20,222 --> 00:20:21,357 PATIENTS AND FAMILIES TO AGREE 587 00:20:21,357 --> 00:20:22,825 TO ENROLL? 588 00:20:22,825 --> 00:20:25,060 AND THEN HOW ARE WE GOING TO BE 589 00:20:25,060 --> 00:20:26,762 ABLE TO GET THEM TO STICK IT OUT 590 00:20:26,762 --> 00:20:27,963 THROUGH THE DURATION OF THE 591 00:20:27,963 --> 00:20:29,698 STUDY? 592 00:20:29,698 --> 00:20:32,468 ONCE THERE ARE RESULTS PATIENT 593 00:20:32,468 --> 00:20:34,370 ORGANIZATIONS CERTAINLY CAN HELP 594 00:20:34,370 --> 00:20:36,038 DISSEMINATE THEM AND ADVOCACY 595 00:20:36,038 --> 00:20:39,541 HERE ENCOMPASSES NOT ONLY 596 00:20:39,541 --> 00:20:40,909 ADDRESSES PATIENTS AND FAMILY 597 00:20:40,909 --> 00:20:43,746 NEEDS IN THEIR EVERYDAY LIFE, 598 00:20:43,746 --> 00:20:46,148 BUT ADVOCACY REGOC POLICY 599 00:20:46,148 --> 00:20:47,783 DECISIONS IMPACTING RARE DISEASE 600 00:20:47,783 --> 00:20:48,817 RESEARCH. 601 00:20:48,817 --> 00:20:50,786 DEPENDING ON TG FOUNDATION, 602 00:20:50,786 --> 00:20:56,392 FUNDING CAN BE PROVIDED AT PD D. 603 00:20:56,392 --> 00:20:56,692 NEXT SLIDE. 604 00:20:56,692 --> 00:20:58,494 NOT ALL FOUNDATIONS ARE EQUAL. 605 00:20:58,494 --> 00:20:59,528 CERTAINLY ONE OF THE MOST 606 00:20:59,528 --> 00:21:01,163 SUCCESSFUL IS THE CF FOUNDATION. 607 00:21:01,163 --> 00:21:03,432 WE HAD A NICE OVERVIEW YESTERDAY 608 00:21:03,432 --> 00:21:04,900 OF THEIR APPROACH SUPPORTING THE 609 00:21:04,900 --> 00:21:05,567 DRUG PIPELINE. 610 00:21:05,567 --> 00:21:09,204 THEY HAVE FUNDING OF EARLY E STE 611 00:21:09,204 --> 00:21:10,372 BIOTECH COMPANIES TO DEVELOP 612 00:21:10,372 --> 00:21:11,573 BREAK THROUGH TREATMENTS. 613 00:21:11,573 --> 00:21:14,610 A ROBUST FUND-RAISING CAPABLE IN 614 00:21:14,610 --> 00:21:17,212 ORDER TO FUND RESEARCH 615 00:21:17,212 --> 00:21:18,180 INITIATIVES BROADLY. 616 00:21:18,180 --> 00:21:19,348 COLLABORATE WELL WITH 617 00:21:19,348 --> 00:21:20,482 PATIENTS,ICALLY NATIONS AND 618 00:21:20,482 --> 00:21:21,650 RESEARCHERS AND HAVE A 619 00:21:21,650 --> 00:21:22,985 COMMITMENT TO ADVOCACY. 620 00:21:22,985 --> 00:21:25,320 SOME ORGANIZATIONS, PARTICULARLY 621 00:21:25,320 --> 00:21:29,224 THOSE THAT MAY BE YOUNGER HAVE 622 00:21:29,224 --> 00:21:31,260 MORE OF A FOCUS ON PATIENT 623 00:21:31,260 --> 00:21:32,261 FAMILY EDUCATION AND SUPPORT. 624 00:21:32,261 --> 00:21:36,331 THEY MAY NOT HAVE THE FUNDING 625 00:21:36,331 --> 00:21:38,100 APPARATUS FOR WIDESPREAD FUNDING 626 00:21:38,100 --> 00:21:38,734 OR RESEARCH. 627 00:21:38,734 --> 00:21:39,268 NEXT SLIDE. 628 00:21:39,268 --> 00:21:46,241 AS YOU START EXPLORING THE 629 00:21:46,241 --> 00:21:47,242 VARIOUS PATIENT FOUNDATIONS OUT 630 00:21:47,242 --> 00:21:48,444 THERE UNDERSTANDING THERE ARE 631 00:21:48,444 --> 00:21:50,646 AREAS OF OVERLAP. 632 00:21:50,646 --> 00:21:53,182 THE FOUNDATION I WORK WITH 633 00:21:53,182 --> 00:21:54,783 SERVES PATIENTS WITH 634 00:21:54,783 --> 00:21:57,419 INTERSTITIAL LUNG DISEASE, BUT 635 00:21:57,419 --> 00:21:59,288 MANY, MANY DIAGNOSES UNDERNEATH 636 00:21:59,288 --> 00:22:00,856 THAT UMBRELLA. 637 00:22:00,856 --> 00:22:03,592 EACH OF THOSE DISORDERS CAN HAVE 638 00:22:03,592 --> 00:22:05,694 POTENTIALLY THEIR OWN 639 00:22:05,694 --> 00:22:05,994 FOUNDATION. 640 00:22:05,994 --> 00:22:13,068 THENEHI RESEARCH FOUNDATION, 641 00:22:13,068 --> 00:22:16,138 COPA SYNDROME FOUNDATION, TBX4 642 00:22:16,138 --> 00:22:17,873 LIFE, PAP DATION. 643 00:22:17,873 --> 00:22:19,308 AMONG THESE YOU HAVENT 644 00:22:19,308 --> 00:22:21,977 POTENTIAL SKILL SETS OR AREAS 645 00:22:21,977 --> 00:22:23,846 THAT CAN HELP FOSTER DIFFERENT 646 00:22:23,846 --> 00:22:25,981 ASPECTS OF THE RESEARCH PROCESS. 647 00:22:25,981 --> 00:22:29,451 SO WORKING WITH THE PATIENT 648 00:22:29,451 --> 00:22:32,554 FOUNDATION OUTSIDE OF THE CS 649 00:22:32,554 --> 00:22:34,289 SPACE IS LIKELY NOT JUST WORKING 650 00:22:34,289 --> 00:22:34,656 WITH ONE. 651 00:22:34,656 --> 00:22:35,390 NEXT SLIDE. 652 00:22:35,390 --> 00:22:37,726 WE TALKED A LITTLE BIT ABOUT HOW 653 00:22:37,726 --> 00:22:41,029 THEY CAN IMPACT MULTIPLE SPOTS 654 00:22:41,029 --> 00:22:44,333 ROCESS OF DRUG 655 00:22:44,333 --> 00:22:44,633 DEVELOPMENT. 656 00:22:44,633 --> 00:22:46,135 AND ONE THING I FAILE TO 657 00:22:46,135 --> 00:22:47,870 MENTION IS THE IMPORTANCE OF 658 00:22:47,870 --> 00:22:51,073 NATURAL HISTORYRY STUDIES AND 659 00:22:51,073 --> 00:22:52,040 UNDERSTAND NATURAL HISTORY OF 660 00:22:52,040 --> 00:22:53,542 DISEASE SOO RESEARCHERS CAN 661 00:22:53,542 --> 00:22:56,345 DESIGN STUDIES WITH APPROPRIATE 662 00:22:56,345 --> 00:22:57,980 END POINTS AND TREATMENT 663 00:22:57,980 --> 00:22:58,614 OUTCOMES. 664 00:22:58,614 --> 00:23:00,582 AGAIN, THE IMPORTANCE OF STUDY 665 00:23:00,582 --> 00:23:02,818 RECRUITMENT AND DESIGN AND 666 00:23:02,818 --> 00:23:04,486 FINALLY DISTRIBUTING RESULTS AND 667 00:23:04,486 --> 00:23:06,855 THEN ALSO THAT POST MARKETING 668 00:23:06,855 --> 00:23:07,756 SURVEILLANCE. 669 00:23:07,756 --> 00:23:09,491 SO PATIENT FOUNDATIONS WILL HAVE 670 00:23:09,491 --> 00:23:10,726 CONNECTIONS WITH FAMILIES ON 671 00:23:10,726 --> 00:23:12,895 THESE MEDICATIONS AND HAVING 672 00:23:12,895 --> 00:23:15,030 MAYBE SIDE EFFECTS OR 673 00:23:15,030 --> 00:23:16,965 EXPERIENCING THINGS POST 674 00:23:16,965 --> 00:23:18,767 MARKETING THAT WILL BE IMPORTANT 675 00:23:18,767 --> 00:23:19,835 AS A COMMUNITY TO UNDERSTAND. 676 00:23:19,835 --> 00:23:23,405 NEXT SLIDE. 677 00:23:23,405 --> 00:23:25,240 SO WE HAVE A NUMBER OF WONDERFUL 678 00:23:25,240 --> 00:23:27,342 SPEAKERS TODAY WHO WILL BE 679 00:23:27,342 --> 00:23:29,611 TOUCHING ON SEVERAL ASPECTS OF 680 00:23:29,611 --> 00:23:32,281 THIS POST IND PHASE OF THE 681 00:23:32,281 --> 00:23:32,514 PROCESS. 682 00:23:32,514 --> 00:23:34,516 WE'LL SPEND TIME LEARNING ABOUT 683 00:23:34,516 --> 00:23:36,018 CHALLENGES TO TRIAL DEVELOPMENT 684 00:23:36,018 --> 00:23:37,252 IN INFANTS AND CHILDREN WITH 685 00:23:37,252 --> 00:23:38,420 RARE LUNG DISEASE. 686 00:23:38,420 --> 00:23:40,589 WE'LL THINK ABOUT HOW TO DESIGN 687 00:23:40,589 --> 00:23:42,357 AND EXECUTE CLINICAL TRIALS, 688 00:23:42,357 --> 00:23:44,693 LEARN FROM THE FIRST CLINICAL 689 00:23:44,693 --> 00:23:48,063 TRIAL IN THE PEDIATRIC 690 00:23:48,063 --> 00:23:50,632 INTERSTITIAL LUNG SPACE, TALK 691 00:23:50,632 --> 00:23:53,302 ABOUT CONTRACTING FOR CLINICAL 692 00:23:53,302 --> 00:23:54,036 STUDIES. 693 00:23:54,036 --> 00:23:56,071 AND IN THE AFTERNOON SUCCESS 694 00:23:56,071 --> 00:23:57,873 STORIES IN THE CLINICAL SPACE. 695 00:23:57,873 --> 00:24:00,309 WE HAVE AN ADULT PERSON WITH CF 696 00:24:00,309 --> 00:24:01,577 SPEAKING ABOUT HIS EXPERIENCE. 697 00:24:01,577 --> 00:24:03,545 WE WILL LEARN ABOUT PCDFOWLKES, 698 00:24:03,545 --> 00:24:05,180 FROM THEM, THEIR EXPERIENCE WITH 699 00:24:05,180 --> 00:24:11,186 OF THE MEDICATIONS FOR PULMONARY 700 00:24:11,186 --> 00:24:12,654 FIBROSIS THAT HAVE BEEN ROBUST 701 00:24:12,654 --> 00:24:14,756 OVER THE PAST FEW YEARS AND 702 00:24:14,756 --> 00:24:21,330 FINALLY LEARN HOW THE LAND 703 00:24:21,330 --> 00:24:23,098 FUNCTION HAS BRUNG ABOUT TARGET 704 00:24:23,098 --> 00:24:26,668 LUNG RESEARCH IN THEIRRC SPACE. 705 00:24:26,668 --> 00:24:26,902 MORNING. 706 00:24:26,902 --> 00:24:29,438 WE WILL BE USING THE Q&A TOOL IN 707 00:24:29,438 --> 00:24:30,539 THE BOTTOM TO ASK QUESTIONS OF 708 00:24:30,539 --> 00:24:32,708 THE PANELISTS. 709 00:24:32,708 --> 00:24:34,509 SO PLEASE KEEP THAT IN MIND AS 710 00:24:34,509 --> 00:24:40,582 QUESTIONS COME TO YOU. 711 00:24:40,582 --> 00:24:43,185 SO WE'LL START WITH OUR SESSION 712 00:24:43,185 --> 00:24:44,853 4, NAVIGATING CLINICAL TRIALS. 713 00:24:44,853 --> 00:24:46,154 AND I GET TO TALK DURING THIS 714 00:24:46,154 --> 00:24:46,755 ONE, TOO. 715 00:24:46,755 --> 00:24:49,758 IF I COULD HAVE THE SPEAKERS POP 716 00:24:49,758 --> 00:24:51,593 THEIR CAMERAS ON SO WE CAN SEE 717 00:24:51,593 --> 00:24:51,927 YOUR FACES. 718 00:24:51,927 --> 00:24:54,830 NEXT SLIDE. 719 00:24:54,830 --> 00:24:58,400 I AM LUCKY TO BE ABLE TO 720 00:24:58,400 --> 00:25:02,304 MODERATE WITH DR. ROBIN 721 00:25:02,304 --> 00:25:05,707 DETERD 722 00:25:05,707 --> 00:25:05,974 DETERDING. 723 00:25:05,974 --> 00:25:07,442 SHE HAS DEDICATED HER RESEARCH 724 00:25:07,442 --> 00:25:08,944 AND CLINICAL CAREER TO CHILDREN 725 00:25:08,944 --> 00:25:10,712 WITH COMPLEX AND RARE LUNG 726 00:25:10,712 --> 00:25:11,046 DISEASE. 727 00:25:11,046 --> 00:25:13,382 FOUNDER OF THE CHILDREN'S 728 00:25:13,382 --> 00:25:14,182 INTERSTITIAL AND DIFFUSE 729 00:25:14,182 --> 00:25:15,984 RESEARCH NETWORK AND A 730 00:25:15,984 --> 00:25:17,552 RECOGNIZED EXPERT IN THE FIELD 731 00:25:17,552 --> 00:25:18,287 OF CHILD. 732 00:25:18,287 --> 00:25:19,955 HEY, ROBIN. 733 00:25:19,955 --> 00:25:20,889 JOINING US THE BEACH, IT 734 00:25:20,889 --> 00:25:27,296 SEEMS. 735 00:25:27,296 --> 00:25:31,266 SO WE'LL GO NEXT SLIDE. 736 00:25:31,266 --> 00:25:31,466 GREAT. 737 00:25:31,466 --> 00:25:36,171 SO OUR OTHER SPEAKERS, ROBIN 738 00:25:36,171 --> 00:25:42,878 GETS, DR. DEET DETERDING. 739 00:25:42,878 --> 00:25:51,720 DR. JENNIFER WAMBACH IEN A 740 00:25:51,720 --> 00:25:52,220 NEONATOLOGIST. 741 00:25:52,220 --> 00:25:55,991 PART OF PEDIATRIC LUNG 742 00:25:55,991 --> 00:26:00,963 TRANSPLANT AND SHE FOCUSES ON 743 00:26:00,963 --> 00:26:03,265 UNDERSTANDING OF INTERSTITIAL 744 00:26:03,265 --> 00:26:05,167 LUNG ZZ AND DEVELOPING 745 00:26:05,167 --> 00:26:05,834 THERAPIES. 746 00:26:05,834 --> 00:26:07,369 DORIS SANCHEZ BRINGS OVER 25 747 00:26:07,369 --> 00:26:08,770 YEARS OF INDUSTRY EXPERIENCE IN 748 00:26:08,770 --> 00:26:15,377 RARE DISEASE AND ONCOLOGY 749 00:26:15,377 --> 00:26:20,282 INDICATIONS.E FOUNDED CLINDEV SI 750 00:26:20,282 --> 00:26:27,622 DR. DETERDING WILL TALK ABOUT 751 00:26:27,622 --> 00:26:31,893 TRIAL AND KAREN SCHNEIDER IS A 752 00:26:31,893 --> 00:26:33,628 BLUEPRINT ATTORNEY AND HELP US 753 00:26:33,628 --> 00:26:34,663 TO THINK ABOUT INVESTIGATOR 754 00:26:34,663 --> 00:26:36,031 INITIATED STUDIES. 755 00:26:36,031 --> 00:26:39,167 I WILL TURN IT OVER TO DR. 756 00:26:39,167 --> 00:26:39,401 WAMBACH. 757 00:26:39,401 --> 00:26:41,136 IF YOU HAVE QUESTIONS, PUT IT IN 758 00:26:41,136 --> 00:26:43,038 THE Q&A TOOL AND WE WILL HAVE 759 00:26:43,038 --> 00:26:45,374 TIME TO TALK WITH OUR PANELISTS 760 00:26:45,374 --> 00:26:52,781 IN THE Q&A DISCUSSION. 761 00:26:52,781 --> 00:26:57,986 >> I'M A NEONATOLOGIST AND TAKE 762 00:26:57,986 --> 00:27:00,989 CARE OF BABIES AND PART OF THE 763 00:27:00,989 --> 00:27:02,391 THE PEDIATRIC LUNG TRANSPLANT 764 00:27:02,391 --> 00:27:02,691 PROGRAM. 765 00:27:02,691 --> 00:27:04,826 I'M TALKING TODAY ABOUT SOME 766 00:27:04,826 --> 00:27:06,294 CLINICAL TRIAL PERSPECTIVES FOR 767 00:27:06,294 --> 00:27:08,730 ENROLLING INFANTS ANDREN 768 00:27:08,730 --> 00:27:13,568 WITH RARE LUNG DISEASES. 769 00:27:13,568 --> 00:27:13,869 NEXT SLIDE. 770 00:27:13,869 --> 00:27:15,670 I HAVE NO RELEVANT DISCLOSURES. 771 00:27:15,670 --> 00:27:18,040 NEXT SLIDE. 772 00:27:18,040 --> 00:27:20,075 SO I'M GOING TO START FIRST WITH 773 00:27:20,075 --> 00:27:22,344 A PATIENT CASE AND THIS IS A 774 00:27:22,344 --> 00:27:24,212 PHONE CALL OR E-MAIL I GET 775 00:27:24,212 --> 00:27:26,181 APPROXIMATELY ONCE A MONTH. 776 00:27:26,181 --> 00:27:29,251 SO THIS WAS A BABY WHO WAS BORN 777 00:27:29,251 --> 00:27:31,253 AT TERM SO 37 WEEKS GESTATION. 778 00:27:31,253 --> 00:27:34,689 SHE IS NOW SIX WEEKS OLD AND HER 779 00:27:34,689 --> 00:27:36,258 CHEST X-RAY IS PICTURED HERE TO 780 00:27:36,258 --> 00:27:37,726 THE RIGHT. 781 00:27:37,726 --> 00:27:39,828 I WAS CONTACTED BECAUSE THIS 782 00:27:39,828 --> 00:27:41,696 BABY HAD SEVERE RESPIRATORY 783 00:27:41,696 --> 00:27:41,930 FAILURE. 784 00:27:41,930 --> 00:27:43,398 BEFORE BIRTH THERE WERE A FEW 785 00:27:43,398 --> 00:27:46,334 HINTS THERE MAY BE ABNORMALITIES 786 00:27:46,334 --> 00:27:52,741 WITH THIS PREGNANCY AND HOW THIS 787 00:27:52,741 --> 00:27:56,111 PREGNANCY WAS DEVELOPING AND THE 788 00:27:56,111 --> 00:28:01,750 TEAM WAS SUSPECTING DUODENAL 789 00:28:01,750 --> 00:28:01,983 ATRESIA. 790 00:28:01,983 --> 00:28:04,286 THE INFANT DEVELOPED RESPIRATORY 791 00:28:04,286 --> 00:28:09,257 ARREST AND PLACED ON CPAP AND 792 00:28:09,257 --> 00:28:12,394 INTUBATED FOR REPAIR OF THE 793 00:28:12,394 --> 00:28:15,063 DUODENAL ATRESIA. 794 00:28:15,063 --> 00:28:19,401 THE INFANT ACUTELY WORSENED AND 795 00:28:19,401 --> 00:28:21,236 HAD SEVERE PULMONARY 796 00:28:21,236 --> 00:28:25,740 HYPERTENSION AND WAS ON ECMO FOR 797 00:28:25,740 --> 00:28:26,208 APPROXIMATELY A WEEK. 798 00:28:26,208 --> 00:28:29,144 NEXT SLIDE. 799 00:28:29,144 --> 00:28:31,947 SO THIS INFANT REMAINS INTUBATED 800 00:28:31,947 --> 00:28:34,182 ON MODERATE VENT LAY TOFR 801 00:28:34,182 --> 00:28:36,251 SETTINGS AT SIXSE WEEKS OF AGE 802 00:28:36,251 --> 00:28:39,754 WITH A SUBSTANTIAL OXYGEN O 803 00:28:39,754 --> 00:28:41,656 REQUIREMENT,ENT 804 00:28:41,656 --> 00:28:44,126 PULMONARY HYPERTENSION, NEAR 805 00:28:44,126 --> 00:28:47,129 SYSTEMIC R.V. PRESSURE AND BEEN 806 00:28:47,129 --> 00:28:53,835 ON MANY VASODILATORS. 807 00:28:53,835 --> 00:28:55,871 GENETIC TESTING WAS SENT SEVERAL 808 00:28:55,871 --> 00:28:57,606 WEEKS AGO WHICH IS STILL 809 00:28:57,606 --> 00:28:57,973 PENDING. 810 00:28:57,973 --> 00:29:01,610 GIVEN THE SEVERITY, THE TEAM 811 00:29:01,610 --> 00:29:04,312 SEND GENOMEEQUENCES RESULTING 812 00:29:04,312 --> 00:29:09,117 IN DENOVO VARIANT IN FOXF1 813 00:29:09,117 --> 00:29:10,819 DISEASE, WHICH MATCHES THIS 814 00:29:10,819 --> 00:29:11,953 PATIENT'S CLINICAL COURSE. 815 00:29:11,953 --> 00:29:14,923 NEXT SLIDE. 816 00:29:14,923 --> 00:29:16,291 SO THE QUESTION FROM THIS 817 00:29:16,291 --> 00:29:17,759 REFERRING POSITIONGS ARE THERE 818 00:29:17,759 --> 00:29:19,394 ANY THERAPIES TO HELP THIS 819 00:29:19,394 --> 00:29:21,530 INFANT? 820 00:29:21,530 --> 00:29:23,698 SO THIS IS JUST A BRIEF OUTLINE 821 00:29:23,698 --> 00:29:25,000 OF MY PRESENTATION TODAY. 822 00:29:25,000 --> 00:29:28,637 I'M GOING TO FOCUS ON AWARENESS 823 00:29:28,637 --> 00:29:31,773 AND RECOGNITION OF THESE RARE 824 00:29:31,773 --> 00:29:33,308 DISORDERS AMONG INFANTS AND 825 00:29:33,308 --> 00:29:34,476 CHILDREN, DIADR APPROACHES 826 00:29:34,476 --> 00:29:36,811 TO IDENTIFYING THE IDEOLOGY FOR 827 00:29:36,811 --> 00:29:37,279 RESPIRATORY FAILURE 828 00:29:37,279 --> 00:29:39,915 N INFANTS 829 00:29:39,915 --> 00:29:40,882 AND CHILDREN. 830 00:29:40,882 --> 00:29:42,150 CURRENTLY AVAILABLECU THERAPIES 831 00:29:42,150 --> 00:29:45,687 WHICH KATELYN MENTIONED ARE 832 00:29:45,687 --> 00:29:47,489 EMPERIC IN LUNG TRANSPLANTATION. 833 00:29:47,489 --> 00:29:49,958 HOW DO WE BRING TRANSLATIONAL 834 00:29:49,958 --> 00:29:51,693 DISCOVERIES THAT ARE EXCITING TO 835 00:29:51,693 --> 00:29:55,297 AFFECTED INFANTS THROUGHOUT 836 00:29:55,297 --> 00:29:57,832 UNITED STATES? 837 00:29:57,832 --> 00:30:02,404 PICTURED HERE, THIS IS A SLIDE 838 00:30:02,404 --> 00:30:05,006 COURTESY OF LARRY NOGEE AND 839 00:30:05,006 --> 00:30:06,808 IDENTIFIED GENES ON THIS SLIDE. 840 00:30:06,808 --> 00:30:08,710 ON THE KPX AX SITUATION YOU SEE 841 00:30:08,710 --> 00:30:15,383 THE AGE OF ON SET AND THE Y AXIS 842 00:30:15,383 --> 00:30:18,486 PRIMARY PHENOTYPES. 843 00:30:18,486 --> 00:30:21,923 THERE HAVE BEEN MARKED ADVANCES 844 00:30:21,923 --> 00:30:23,758 OF LUNG DISEASES IN INFANTS, 845 00:30:23,758 --> 00:30:27,095 CHILDREN AND ADULTS. 846 00:30:27,095 --> 00:30:32,234 THE PHENOTYPES HAVE SOME OVERLAP 847 00:30:32,234 --> 00:30:33,635 LISTED ON THE Y AXIS. 848 00:30:33,635 --> 00:30:36,605 SOME INFANTS ARE PRESENTING WITH 849 00:30:36,605 --> 00:30:38,440 SEVERE DIFFUSE LUNG DIAGNOSE AND 850 00:30:38,440 --> 00:30:40,308 PROGRESSIVE FIBROSIS, OTHERS 851 00:30:40,308 --> 00:30:45,947 HAVE GROWTH ABNORMALITIES AND 852 00:30:45,947 --> 00:30:47,449 OTHERING PRESENTING WITH 853 00:30:47,449 --> 00:30:49,317 PULMONARY HYPERTENSION OR 854 00:30:49,317 --> 00:30:51,686 INFECTIOUS OR IMMUNE PHENOTYPES. 855 00:30:51,686 --> 00:30:53,555 FOR THE PURPOSES OF DISCUSSION 856 00:30:53,555 --> 00:30:55,423 TODAY WE ARE GOING TO FOCUS ON 857 00:30:55,423 --> 00:30:58,493 THESE FOUR GENES AS THESE 858 00:30:58,493 --> 00:30:59,794 INFANTS WITH PATHOGENIC VARNTSS 859 00:30:59,794 --> 00:31:01,596 IN THESE GENES HAVE SEVERE 860 00:31:01,596 --> 00:31:04,833 RESPIRATORY FAILURE AND THERE 861 00:31:04,833 --> 00:31:07,435 ARE PRECLINICAL STUDIES THAT ARE 862 00:31:07,435 --> 00:31:09,938 EXCITING AND ENCOURAGING THAT 863 00:31:09,938 --> 00:31:11,773 POTENTIALLY COULD DEVELOP 864 00:31:11,773 --> 00:31:14,976 THERAPIES FOR THESE INFANTS. 865 00:31:14,976 --> 00:31:17,312 SO AGAIN BACK TO THE AWARENESS 866 00:31:17,312 --> 00:31:19,648 AND RECOGNITION OF THESE 867 00:31:19,648 --> 00:31:19,914 DISORDERS. 868 00:31:19,914 --> 00:31:20,115 SO T 869 00:31:20,115 --> 00:31:22,450 MOST COMMON CAUSES OF 870 00:31:22,450 --> 00:31:24,552 RESPIRATORY FAILURE, GENETIC 871 00:31:24,552 --> 00:31:27,856 RESPIRATORY FAILURE IN TERM 872 00:31:27,856 --> 00:31:30,625 INFANTS ARE GENETIC DISORDERS 873 00:31:30,625 --> 00:31:40,201 ARE SURFACTANT, VARIANTS, ABCA3 874 00:31:40,201 --> 00:31:43,338 DEFICIENCY AND NKX 2RKS-1. 875 00:31:43,338 --> 00:31:44,906 THESE ARE IMPORTANT FOR 876 00:31:44,906 --> 00:31:49,944 METABOLISM IN THE TYPE 2 CELL. 877 00:31:49,944 --> 00:31:54,983 SF PROTEIN B AND KR. 878 00:31:54,983 --> 00:31:57,652 ABCA3 SIT ON THE BODIES THAT 879 00:31:57,652 --> 00:32:00,722 KATELYN DESCRIBED ON THE IMAGE 880 00:32:00,722 --> 00:32:04,993 FROM HER PATIENT. 881 00:32:04,993 --> 00:32:09,331 AND ABCA3 COMBINE TO MAKE 882 00:32:09,331 --> 00:32:14,869 SURFACTANT. 883 00:32:14,869 --> 00:32:17,472 THE TRANSCRIPTION FACTOR IS 884 00:32:17,472 --> 00:32:18,239 IMPORTANT FOR LUNG DEVELOPMENT 885 00:32:18,239 --> 00:32:20,575 BUT ALSO FOR DEVELOPMENT OF THE 886 00:32:20,575 --> 00:32:21,443 THYROID AND BRAIN. 887 00:32:21,443 --> 00:32:25,914 THE PATIENT DESCR IN MY 888 00:32:25,914 --> 00:32:29,651 VIGNETTE AT ACDMPC. 889 00:32:29,651 --> 00:32:32,754 THIS IS DUE TO PATHOGENIC 890 00:32:32,754 --> 00:32:35,223 VARIANTS IN FOX F1 THAT 891 00:32:35,223 --> 00:32:37,859 INCORPORATE THE GENE OR UPSTREAM 892 00:32:37,859 --> 00:32:39,994 REGULATORY REGIONS. 893 00:32:39,994 --> 00:32:42,931 WITH INCREASED USE OF GENE 894 00:32:42,931 --> 00:32:44,232 SEQUENCING WE ARE IDENTIFIES 895 00:32:44,232 --> 00:32:46,267 OTHER CAUSES OF SEVERE LUNG 896 00:32:46,267 --> 00:32:47,902 INFANT AND CHI 897 00:32:47,902 --> 00:32:52,674 AND MANY DISORDERS HAVE 898 00:32:52,674 --> 00:32:53,007 ABNORMALITIES. 899 00:32:53,007 --> 00:32:55,243 WHETHER COPA OR STING OR 900 00:32:55,243 --> 00:32:59,481 MULTIORGAN SYSTEM INVOLVEMENT.YS 901 00:32:59,481 --> 00:33:02,751 SO WHEN TO SUSPECT THESE 902 00:33:02,751 --> 00:33:03,017 DISORDERS? 903 00:33:03,017 --> 00:33:05,987 A TERM INFANT WITH PERSISTENT 904 00:33:05,987 --> 00:33:07,489 RESPIRATORY FAILURE MORE THAN 905 00:33:07,489 --> 00:33:08,790 SEVEN DAYS SHOULD INITIATE 906 00:33:08,790 --> 00:33:11,860 CONCERN FOR A RARE GENETIC 907 00:33:11,860 --> 00:33:12,560 CONDITION AFFECTING LUNG 908 00:33:12,560 --> 00:33:14,662 DEVELOPMENT AND LUNG FUNCTION. 909 00:33:14,662 --> 00:33:17,132 CERTAINLY WE KNOW THERE ARE RISK 910 00:33:17,132 --> 00:33:19,367 FACTORS FOR TERM BABIES TO HAVE 911 00:33:19,367 --> 00:33:21,736 BREATHING PROBLEMS AFTER BIRTH, 912 00:33:21,736 --> 00:33:23,705 INCLUDING 37 WEEKS VERSUS 40 913 00:33:23,705 --> 00:33:24,239 WEEKS. 914 00:33:24,239 --> 00:33:25,874 THERE IS A RANGE OF BEING 915 00:33:25,874 --> 00:33:28,276 CONSIDERED TERM AND THOSE IN THE 916 00:33:28,276 --> 00:33:30,945 EARLIER SPECTRUM CAN HAVE 917 00:33:30,945 --> 00:33:32,180 RESPIRATORY DISTRESS THAT 918 00:33:32,180 --> 00:33:34,249 IMPROVING IN THE FIRST WEEK OF 919 00:33:34,249 --> 00:33:35,550 LIFE. 920 00:33:35,550 --> 00:33:39,354 INFANTS BORN TO WOMEN WHO HAVEE 921 00:33:39,354 --> 00:33:44,192 DIE BEES, MALE SEX, DESCENT. 922 00:33:44,192 --> 00:33:46,494 ALL OF THOSE ARE RISK FACTORS 923 00:33:46,494 --> 00:33:47,896 FOR RESPIRATORY DISTRESS IN THE 924 00:33:47,896 --> 00:33:50,965 FIRST FEW DAYS OF LIFE THAT 925 00:33:50,965 --> 00:33:51,966 TYPICALLY SHOULD IMPROVE BY THE 926 00:33:51,966 --> 00:33:54,035 END OF THE FIRST WEEK. 927 00:33:54,035 --> 00:33:55,737 IF AN INFANT PRESENTS WITHOUT 928 00:33:55,737 --> 00:33:57,472 THOSE RISK FACTORS IT SHOULD 929 00:33:57,472 --> 00:34:00,942 FLAG THE ATTENTION TO SUSPECT AN 930 00:34:00,942 --> 00:34:01,509 UNDERLYING CONDITION. 931 00:34:01,509 --> 00:34:04,045 IF THERE IS A POSITIVE FAMILY 932 00:34:04,045 --> 00:34:07,715 HISTORY MANY DISORDERS ARE 933 00:34:07,715 --> 00:34:12,554 AUTOSORKS SOMAL RECESSSIVE. 934 00:34:12,554 --> 00:34:16,057 THERE ARE LIMITED PRENATAL 935 00:34:16,057 --> 00:34:18,226 FINDINGS WITH THE EXCEPTION OF 936 00:34:18,226 --> 00:34:26,801 ACDMPV. 937 00:34:26,801 --> 00:34:28,870 SO SOMETIMES FETUSES WILL BE 938 00:34:28,870 --> 00:34:31,272 IDENTIFIED WITH ABNORMALITIES IN 939 00:34:31,272 --> 00:34:34,776 THESE ORGAN SYSTEMS PROMPTINGMS 940 00:34:34,776 --> 00:34:36,945 PRENATAL GENETIC TESTIN 941 00:34:36,945 --> 00:34:42,383 FOR THE GENETIC SURFACCTANT 942 00:34:42,383 --> 00:34:50,258 DISORD TLERS ARE NO OTHERER ENO. 943 00:34:50,258 --> 00:34:53,661 IN KH2-1 IS IMPORTANT FOR LUNG 944 00:34:53,661 --> 00:34:54,963 DEVELOPMENT AND THE THYROID AND 945 00:34:54,963 --> 00:34:57,232 THE SPRAIN AND OTHER PHENOTYPES 946 00:34:57,232 --> 00:34:59,067 MAY BE PRESENT IN THE INFANT WHO 947 00:34:59,067 --> 00:35:04,405 HAS A PATHOGENIC VARIANT IN 948 00:35:04,405 --> 00:35:04,639 NKX2-1. 949 00:35:04,639 --> 00:35:06,508 PART OF THE CHALLENGE IS THESE 950 00:35:06,508 --> 00:35:08,376 ARE VERY RARE CONDITIONS. 951 00:35:08,376 --> 00:35:12,714 MOST NEONATOLOGISTS WILL SEE ONE 952 00:35:12,714 --> 00:35:14,215 OR TWO OF THESE BABIES IN THE 953 00:35:14,215 --> 00:35:22,190 SPAN OF THEIR CAREER. 954 00:35:22,190 --> 00:35:25,660 SURFACTANT PROSTEEN D DEFICIENCD 955 00:35:25,660 --> 00:35:25,894 IS RARE. 956 00:35:25,894 --> 00:35:28,029 THIS IS ANYWHERE FROM 1 TO 4 957 00:35:28,029 --> 00:35:30,164 INFANTS TOTAL IN THE UNITED 958 00:35:30,164 --> 00:35:37,405 STATES BORN WITH SPV. 959 00:35:37,405 --> 00:35:40,575 LOOKING AT THE FREE KWENS OF LOS 960 00:35:40,575 --> 00:35:42,977 OF VARIANCE IN NOMAD A LARGE 961 00:35:42,977 --> 00:35:44,746 DATA BASE FROM APPROXIMATELY 962 00:35:44,746 --> 00:35:46,247 800,000 ADULT INDIVIDUALS, IF 963 00:35:46,247 --> 00:35:48,816 YOU LOOK AT THE LOSS OFION 964 00:35:48,816 --> 00:35:50,451 VARIANTS AND THE KNOWNWN 965 00:35:50,451 --> 00:35:52,587 PATHOGENIC VAMPBTS WE ESTIMATE 966 00:35:52,587 --> 00:35:55,523 DISEASE INCIDENCE OF TO 45,000 967 00:35:55,523 --> 00:35:56,324 BIRTHS. 968 00:35:56,324 --> 00:35:58,059 SO ABOUT 100 INFANTS PER YEAR. 969 00:35:58,059 --> 00:35:59,794 THIS IS CERTAINLY AN 970 00:35:59,794 --> 00:36:01,062 UNDERESTIMATE AS MOST OF THE 971 00:36:01,062 --> 00:36:04,065 VARIANTS THAT RESULT IN ABCA3 972 00:36:04,065 --> 00:36:05,733 DEFICIENCY ARE PRIVATE AND 973 00:36:05,733 --> 00:36:07,468 UNIQUE TO INDIVIDUALS AND 974 00:36:07,468 --> 00:36:07,936 FAMILIES AND 975 00:36:07,936 --> 00:36:10,305 OUR ABILITY TO 976 00:36:10,305 --> 00:36:11,739 PREDICT IS MORE LIMITED. 977 00:36:11,739 --> 00:36:14,175 AND IT IS REALLY DIFFICULT TO 978 00:36:14,175 --> 00:36:23,117 ESTIMATE THE INCIDENCE OF 979 00:36:23,117 --> 00:36:24,552 ALVEOLAR CAPILLARY. 980 00:36:24,552 --> 00:36:26,321 WE SEE PEN ATLANTAS AMONG 981 00:36:26,321 --> 00:36:29,657 FAMILIES WITH THE SAME 982 00:36:29,657 --> 00:36:31,259 PATHOGENIC VARIANT THERE CAN BE 983 00:36:31,259 --> 00:36:32,060 DIFFERENCES IN AGE OF 984 00:36:32,060 --> 00:36:33,595 PRESENTATION AND SEVERITY OF 985 00:36:33,595 --> 00:36:36,931 DISEASE AND PROGRESSION. 986 00:36:36,931 --> 00:36:37,231 NEXT SLIDE. 987 00:36:37,231 --> 00:36:38,600 ISOS REALLYOSIS 988 00:36:38,600 --> 00:36:43,738 MADE WITH GENETIC TESTING AND 989 00:36:43,738 --> 00:36:46,941 TREE OWGS IS PREFERRED OVER 990 00:36:46,941 --> 00:36:49,110 PANEL TESTING. 991 00:36:49,110 --> 00:36:52,280 TREE OWHOLE GENOME SEQUENCE 992 00:36:52,280 --> 00:36:54,048 GIVES US COPY NUMBER VARIANCE 993 00:36:54,048 --> 00:36:56,084 IMPORTANT TO RARE GENETIC LUNG 994 00:36:56,084 --> 00:36:56,351 DISEASES. 995 00:36:56,351 --> 00:36:58,753 THE TURN AROUND TIME IS MUCH 996 00:36:58,753 --> 00:36:59,387 SHORTER. 997 00:36:59,387 --> 00:37:01,255 RIGHT NOW WGS INITIAL RESULTS 998 00:37:01,255 --> 00:37:03,424 ARE BACK IN SEVEN DAYS AND THE 999 00:37:03,424 --> 00:37:06,361 FINAL REPORT IS 14 DAYS AND I 1000 00:37:06,361 --> 00:37:07,862 EXPECT THAT THIS WILL SHORTEN 1001 00:37:07,862 --> 00:37:09,197 WITHWITIME. 1002 00:37:09,197 --> 00:37:11,099 AND TREE E GENOME 1003 00:37:11,099 --> 00:37:13,735 SEQUENCINGEN GIVES US THE ABILI 1004 00:37:13,735 --> 00:37:16,804 TONTIFY DENOVO VARIANTS NNT 1005 00:37:16,804 --> 00:37:19,140 PRESENT IN THE PARENTS AND PHASE 1006 00:37:19,140 --> 00:37:19,407 VARIANTS. 1007 00:37:19,407 --> 00:37:25,446 THIS IS IMPORTANT FOR ABCA3 AS 1008 00:37:25,446 --> 00:37:28,349 WE HAVE FOUND THEM OCCUR ON 1009 00:37:28,349 --> 00:37:28,950 CYSTS. 1010 00:37:28,950 --> 00:37:32,520 IF THERE ARE TWOABCA3 VARIANTS 1011 00:37:32,520 --> 00:37:34,188 IDENTIFIED IT IS IMPORTANT I TO 1012 00:37:34,188 --> 00:37:36,357 HAVE THE PARENTAL SAMPLES TO 1013 00:37:36,357 --> 00:37:38,559 DETERMINE IF ONE OR BOTH ALLELES 1014 00:37:38,559 --> 00:37:40,128 ARE AFFECTED. 1015 00:37:40,128 --> 00:37:47,035 IN THE NICU THERE ARE TRIALS IN 1016 00:37:47,035 --> 00:37:48,102 FOR CL ILL INFANTS. 1017 00:37:48,102 --> 00:37:49,837 I HAVE A COUPLE OF STUDIES 1018 00:37:49,837 --> 00:37:51,706 LISTED TO THE RIGH 1019 00:37:51,706 --> 00:37:53,007 TI FOR A BABY 1020 00:37:53,007 --> 00:37:57,178 IN THE NICU SUSPECTED OF HAVING 1021 00:37:57,178 --> 00:38:00,214 A GENETIC CONDITION IS ABOUT 30% 1022 00:38:00,214 --> 00:38:00,515 TO 35%. 1023 00:38:00,515 --> 00:38:04,585 NEXT SLIDE. 1024 00:38:04,585 --> 00:38:07,655 HOWEVER, WE HAVE CONSIDERABLE 1025 00:38:07,655 --> 00:38:09,691 VARIATION IN THE AVAILABILITY OF 1026 00:38:09,691 --> 00:38:15,163 GENETIC TESTING BY DIFFERENT 1027 00:38:15,163 --> 00:38:15,963 NICUs. 1028 00:38:15,963 --> 00:38:19,133 THIS IS A STUDY DON'T LOOKING AT 1029 00:38:19,133 --> 00:38:23,438 SERVICE AND MULTIPLE LEVEL 1030 00:38:23,438 --> 00:38:26,741 4NICUs THROUGHOUT THE UNITED 1031 00:38:26,741 --> 00:38:27,208 STATES. 1032 00:38:27,208 --> 00:38:29,711 THERE IS VARIABILITY OF 1033 00:38:29,711 --> 00:38:32,580 GENETICISTS, GENETIC COUNSELING 1034 00:38:32,580 --> 00:38:33,948 SE THE APPROVAL 1035 00:38:33,948 --> 00:38:37,985 PROCESS FOR GENOME SEQUENCING. 1036 00:38:37,985 --> 00:38:40,121 LUNG BIOPSY, WE ARE ASKED IF 1037 00:38:40,121 --> 00:38:41,656 THIS IS NECESSARY FOR THESE 1038 00:38:41,656 --> 00:38:43,991 INFANTS AND IF A PATHOGENIC 1039 00:38:43,991 --> 00:38:48,229 VARIANT IS IDENTIFIED WITH ME 1040 00:38:48,229 --> 00:38:52,300 SEQUENCING, GENOME SEQUENCES OR 1041 00:38:52,300 --> 00:38:53,935 WITH THE CLINICAL COURSE THIS 1042 00:38:53,935 --> 00:38:56,170 CAN BE DIAGNOSTIC AND THE CHILD 1043 00:38:56,170 --> 00:38:58,606 DOES NOT NECESSARILY NEED A LUNG 1044 00:38:58,606 --> 00:38:58,873 BIOPSY. 1045 00:38:58,873 --> 00:39:00,608 THERE ARE SITUATIONS WHERE A 1046 00:39:00,608 --> 00:39:03,478 LUNG BIOPSY IS HELPFUL. 1047 00:39:03,478 --> 00:39:06,280 THIS COULD HAPPEN WHEN THE OTHER 1048 00:39:06,280 --> 00:39:09,917 RESULTS ARE NONDIAGNOSTIC. 1049 00:39:09,917 --> 00:39:12,253 VARIANTS ARE IDENTIFIED, THE 1050 00:39:12,253 --> 00:39:14,255 CHILD AS AN ATYPICAL COURSE AND 1051 00:39:14,255 --> 00:39:16,691 TO EXCLUDE OTHERDISORDERS THAT 1052 00:39:16,691 --> 00:39:22,096 MAY IMPROVE WITH TIME, PODIUM 1053 00:39:22,096 --> 00:39:24,999 NOIR INTERSTITIAL GLYCOGENOS 1054 00:39:24,999 --> 00:39:26,067 SIXTH. 1055 00:39:26,067 --> 00:39:34,008 THERE IS AIR LEAGUE OR HEMO 1056 00:39:34,008 --> 00:39:34,776 THORAX. 1057 00:39:34,776 --> 00:39:38,780 IT CAN EXACERBATE PULMONARY 1058 00:39:38,780 --> 00:39:40,114 HYPERTENSION. 1059 00:39:40,114 --> 00:39:41,682 THE LUNG BIOPSY IS VERY HELPFUL, 1060 00:39:41,682 --> 00:39:45,787 THERE TR A LOT OF ORR LAPPING 1061 00:39:45,787 --> 00:39:47,455 HISTOLOGIC FEATURES. 1062 00:39:47,455 --> 00:39:51,159 PICTURED ON THE BOTTOM LEFT LUNG 1063 00:39:51,159 --> 00:39:55,997 BIOPSY RESULTS IMAGES FROM 1064 00:39:55,997 --> 00:39:59,634 INFANTS WITH DEFICIENCIES AND. 1065 00:39:59,634 --> 00:40:03,070 AND YOU CAN SEE THAT MANY OF 1066 00:40:03,070 --> 00:40:06,073 THESE DISORDERS HAVE TYPE TWO 1067 00:40:06,073 --> 00:40:11,112 CELL HYPERPLASIA. 1068 00:40:11,112 --> 00:40:13,848 SO WHILE THESE LUNG BIOPSIES MAY 1069 00:40:13,848 --> 00:40:24,425 BE HELPFUL POINTING US IN 1070 00:40:24,425 --> 00:40:25,693 SURFACTANT THEY DON'T 1071 00:40:25,693 --> 00:40:29,630 DISTINGUISH WITH THE IDEOLOGIES. 1072 00:40:29,630 --> 00:40:32,366 ABCA3 DEFICIENCY THERE ARE 1073 00:40:32,366 --> 00:40:34,202 CHARACTERISTIC CHANGES IN THE 1074 00:40:34,202 --> 00:40:34,602 STRUCTURE. 1075 00:40:34,602 --> 00:40:38,105 IF A LUNG BIOPSY IS OBTAINED WE 1076 00:40:38,105 --> 00:40:43,911 HIGHLY ENCOURAGEENNI 1077 00:40:43,911 --> 00:40:45,046 ELECTROMICROSCOPY THAT CAN BE 1078 00:40:45,046 --> 00:40:46,848 HELPFUL IN THE DIAGNOSIS. 1079 00:40:46,848 --> 00:40:50,117 I WILL HIGHLIGHT AN ARTICLE 1080 00:40:50,117 --> 00:40:54,155 PUBLISHED BY THE GROUP FRO 1081 00:40:54,155 --> 00:40:57,191 TORONTO WHO DESCRIBE MORTALITY 1082 00:40:57,191 --> 00:40:59,360 OF PEDIATRIC SURGICAL LUNG 1083 00:40:59,360 --> 00:40:59,861 BIOPSIES. 1084 00:40:59,861 --> 00:41:02,463 THIS WAS A GREAT DESCRIPTION OF 1085 00:41:02,463 --> 00:41:04,699 ALL OF THE SERIES ASSOCIATED 1086 00:41:04,699 --> 00:41:06,200 MORBIDITIES WITH LUNG BIOPSY. 1087 00:41:06,200 --> 00:41:10,404 NEXT SLIDE. 1088 00:41:10,404 --> 00:41:12,540 SO IT IS VERY IMPORTANT AS WE 1089 00:41:12,540 --> 00:41:14,208 THINK ABOUT CURRENT THERAPIES TO 1090 00:41:14,208 --> 00:41:16,944 UNDERSTAND THE GENE TYPE 1091 00:41:16,944 --> 00:41:17,912 PHENOTYPE CORRELATION. 1092 00:41:17,912 --> 00:41:19,513 THERE ARE SOME DISORDERS THAT 1093 00:41:19,513 --> 00:41:21,983 ARE LETHAL WITHIN THE FIRST FEW 1094 00:41:21,983 --> 00:41:24,852 MONTHS OF LIFE WITHOUT LUNG 1095 00:41:24,852 --> 00:41:25,219 TRANSPLANTATION. 1096 00:41:25,219 --> 00:41:34,161 WE THINK SURFACTANT PROTEIN B 1097 00:41:34,161 --> 00:41:34,795 DEFICIENT. 1098 00:41:34,795 --> 00:41:37,298 THIS IS THE MOST COMMON IDEOLOGY 1099 00:41:37,298 --> 00:41:39,333 OF THE DEFICIENCY. 1100 00:41:39,333 --> 00:41:39,800 WE 1101 00:41:39,800 --> 00:41:44,038 OCCASIONAL LONG-TERM LSURVIVOR 1102 00:41:44,038 --> 00:41:44,305 DESCRIBED. 1103 00:41:44,305 --> 00:41:48,276 THESE INDIVIDUALS TYPICALLY HAVE 1104 00:41:48,276 --> 00:41:50,578 ONE VARIANT THAT PERMITS SOME 1105 00:41:50,578 --> 00:41:53,714 AMOUNT OF SURFACTANT PROTEIN B 1106 00:41:53,714 --> 00:41:57,251 FUNCTION. 1107 00:41:57,251 --> 00:41:59,520 SOME DIE WITHIN THE FEW FEW 1108 00:41:59,520 --> 00:42:01,455 MONTHS OF LIFE WITHOUT LUNG 1109 00:42:01,455 --> 00:42:03,958 TRANSPLANTATION. 1110 00:42:03,958 --> 00:42:11,565 AL B VEOLAR -- SHORTLY AFTER 1111 00:42:11,565 --> 00:42:13,501 BIRTH AND DEATH WITHIN THE FIRST 1112 00:42:13,501 --> 00:42:16,370 FEW MONTHS OF LIFE WITHOUT LUNG 1113 00:42:16,370 --> 00:42:16,737 TRANSPLANTATION. 1114 00:42:16,737 --> 00:42:20,508 WE HAVE INCREASED OUR USE OF 1115 00:42:20,508 --> 00:42:23,811 GENETIC TESTING AMONG CHILDREN 1116 00:42:23,811 --> 00:42:25,746 WITH INTERSTITIAL LUNG DISEASE, 1117 00:42:25,746 --> 00:42:28,649 WE HAVE FOUND SOME OLDER INFANTS 1118 00:42:28,649 --> 00:42:32,653 AND CHILDREN WHO HAVE PATHOGE 1119 00:42:32,653 --> 00:42:38,826 IC VARIANTS IN FOX F1 WHO HAVE 1120 00:42:38,826 --> 00:42:40,428 BEEN ABLE TO SURVIVE. 1121 00:42:40,428 --> 00:42:43,331 IT SEEMS THOSE INDIVIDUALS HAVE 1122 00:42:43,331 --> 00:42:44,065 PATCHY INVOLVEMENT OF DISEASE 1123 00:42:44,065 --> 00:42:45,333 THROUGHOUT THEIR LUNGS AND THE 1124 00:42:45,333 --> 00:42:47,768 REASONS ARE NOT ENTIRELY CLEAR. 1125 00:42:47,768 --> 00:42:50,738 SO IN TALKING ABOUT THOSE WHO 1126 00:42:50,738 --> 00:42:54,041 HAVE PRETTY CLEVE CUT GENE TYPE 1127 00:42:54,041 --> 00:42:58,079 PHENOTYPE CORRELATIONS, THERE 1128 00:42:58,079 --> 00:43:02,316 ARE THOSE WITH MORE VARIABLE 1129 00:43:02,316 --> 00:43:04,085 VARIANTS. 1130 00:43:04,085 --> 00:43:09,056 THOSE WITH ABCA3 DEFICIENCY 1131 00:43:09,056 --> 00:43:11,826 AND -- SOME DELETIONS THAT MAY 1132 00:43:11,826 --> 00:43:13,928 PERMIT SOME AMOUNTOM OF FUNCTIO. 1133 00:43:13,928 --> 00:43:14,962 THESE FAMILIES ARE MORE 1134 00:43:14,962 --> 00:43:17,365 DIFFICULT TO COUNCIL INOU TERMSF 1135 00:43:17,365 --> 00:43:19,333 THE DISEASE PROGRESSION AND 1136 00:43:19,333 --> 00:43:20,334 PREDICTION FOR SURVIVAL. 1137 00:43:20,334 --> 00:43:22,370 AND, AGAIN, CERTAINLY WE ARE 1138 00:43:22,370 --> 00:43:24,305 IDENTIFYING OTHER INDIVIDUALS 1139 00:43:24,305 --> 00:43:27,274 WHO HAVE MONOGENIC CAUF 1140 00:43:27,274 --> 00:43:29,310 LUAUEASE IN INFANTS AND 1141 00:43:29,310 --> 00:43:29,577 CHILDREN. 1142 00:43:29,577 --> 00:43:32,079 THESE ARE OFTEN ULTRA RARE 1143 00:43:32,079 --> 00:43:34,448 DISORDERS AND WE KNOW LESS ABOUT 1144 00:43:34,448 --> 00:43:36,984 THE NATURAL HISTORY OF THESE 1145 00:43:36,984 --> 00:43:38,185 DISORDERS WE NEED TO FOLLOW 1146 00:43:38,185 --> 00:43:39,653 THESE INFANTS AND CHILDREN OVER 1147 00:43:39,653 --> 00:43:39,854 TIME. 1148 00:43:39,854 --> 00:43:42,923 NEXT SLIDE. 1149 00:43:42,923 --> 00:43:44,925 SO OUR CURRENT THERAPIES, 1150 00:43:44,925 --> 00:43:46,727 MEDICAL THERAPIES ARE QUITE 1151 00:43:46,727 --> 00:43:46,961 LIMITED. 1152 00:43:46,961 --> 00:43:49,764 THEY REALLY INCLUDE 1153 00:43:49,764 --> 00:43:52,633 ANTI-INFLAMMATORY MEDICATIONS 1154 00:43:52,633 --> 00:43:56,804 GIVEN EM P TO CHILDREN 1155 00:43:56,804 --> 00:44:00,141 WITH INTERSTITIAL LUNG DISEASES. 1156 00:44:00,141 --> 00:44:02,510 TRANSPLANT IS THE DEFINITIVE 1157 00:44:02,510 --> 00:44:03,744 TREATMENT OPTION FOR PROGRESSIVE 1158 00:44:03,744 --> 00:44:06,514 RESPIRATORY FAILURE. 1159 00:44:06,514 --> 00:44:07,982 PICTURED HERE ARE FIVE-YEAR 1160 00:44:07,982 --> 00:44:10,251 SURVIVAL RATES FOR INFANTS AND 1161 00:44:10,251 --> 00:44:10,885 CHILDREN. 1162 00:44:10,885 --> 00:44:13,721 ON THE KPX AXIS IS TIME IN 1163 00:44:13,721 --> 00:44:16,123 MONTHS AFTER TRANSPLANT, Y IS 1164 00:44:16,123 --> 00:44:16,824 SURVIVAL PROBABILITY. 1165 00:44:16,824 --> 00:44:18,859 FOR INFANTS FIVE YEAR SURVIVAL 1166 00:44:18,859 --> 00:44:21,762 IS 60%, FOR CHILDREN 80%. 1167 00:44:21,762 --> 00:44:24,665 THESE OUTCOMES HAVE BEEN 1168 00:44:24,665 --> 00:44:25,566 RELATIVELY STABLE, MAYBE 1169 00:44:25,566 --> 00:44:28,135 SOMEWHAT IMPROVED OVER THE PAST 1170 00:44:28,135 --> 00:44:31,005 THREE DEKCADES. 1171 00:44:31,005 --> 00:44:33,874 INFANTS ARE VERY HILL AT TIME OF 1172 00:44:33,874 --> 00:44:35,276 TRANSPLANT, HIGH RISK FOR 1173 00:44:35,276 --> 00:44:38,345 DEVELOPMENTAL DELAYS, HEARING 1174 00:44:38,345 --> 00:44:41,816 LOSS, NAL 1175 00:44:41,816 --> 00:44:46,353 SUPPLEMENTATION AND NDBRONCHULIS 1176 00:44:46,353 --> 00:44:50,224 IS A MAJOR MORBIDITY O A 1177 00:44:50,224 --> 00:44:53,227 SLIGHTLY LOWER RISK IN INFANTS 1178 00:44:53,227 --> 00:44:56,030 MAYBE TAKING ADVANTAGE OF THEIR 1179 00:44:56,030 --> 00:44:59,333 NAIVE IMMUNE SYSTEM. 1180 00:44:59,333 --> 00:44:59,633 NEXT SLIDE. 1181 00:44:59,633 --> 00:45:01,702 SO HOW TO DRINK THESE 1182 00:45:01,702 --> 00:45:03,604 TRANSLATIONAL DISCOVERYS TO 1183 00:45:03,604 --> 00:45:04,705 INFANTS. 1184 00:45:04,705 --> 00:45:05,339 CHALLENGES. 1185 00:45:05,339 --> 00:45:08,843 INFANTS OFTEN IDENTIFIED AT 4 TO 1186 00:45:08,843 --> 00:45:12,613 6 WEEKS OF AGE, EXPOSED TO 1187 00:45:12,613 --> 00:45:14,148 MECHANICAL VENTILATION AND HAVE 1188 00:45:14,148 --> 00:45:15,516 INJURE TO T NGS. 1189 00:45:15,516 --> 00:45:17,818 THEY ARE PRESENT AT CENTERS 1190 00:45:17,818 --> 00:45:27,428 THROUGHOUT THE UNITED STATES, . 1191 00:45:27,428 --> 00:45:29,563 THEY ARE CRITICALLY ILL AND DO 1192 00:45:29,563 --> 00:45:32,633 NOT DO A GOOD JOB -- PRETERM 1193 00:45:32,633 --> 00:45:35,469 INFANTS ARE MORE LIKELY TO HAVE 1194 00:45:35,469 --> 00:45:38,939 RESPIRATORY DISTRESS DUE TO 1195 00:45:38,939 --> 00:45:45,112 DEVELOPMENTALCIENT SI OFI 1196 00:45:45,112 --> 00:45:48,616 SURFACTANT PRODUCTION. 1197 00:45:48,616 --> 00:45:49,984 AND THINKING ABOUT BRINGING 1198 00:45:49,984 --> 00:45:50,451 THERAPIES WHICH 1199 00:45:50,451 --> 00:45:51,752 INFANTS AND 1200 00:45:51,752 --> 00:45:53,120 CHILDREN SHOULD WE TARGET? 1201 00:45:53,120 --> 00:45:55,990 SHOULD WE TARGET THOSE WITH THE 1202 00:45:55,990 --> 00:46:00,127 MOST SEVERE PHENOTYPES UNLIKELY 1203 00:46:00,127 --> 00:46:02,096 TO SURVIVE WITHOUT LUNG 1204 00:46:02,096 --> 00:46:03,664 TRANSPLANTATION OR CHILDREN WITH 1205 00:46:03,664 --> 00:46:06,500 INTERSTITIAL LUNG DISEASE AND 1206 00:46:06,500 --> 00:46:08,202 MORE CHRONIC LUNG DISEASE? 1207 00:46:08,202 --> 00:46:09,303 THAT IS AN IMPOR 1208 00:46:09,303 --> 00:46:11,105 CONSIDERATION ASIO WE THINK ABO 1209 00:46:11,105 --> 00:46:12,206 THESE THERAPIES. 1210 00:46:12,206 --> 00:46:14,208 CERTAINLY ANY THERAPY WE HAVE 1211 00:46:14,208 --> 00:46:18,412 HAS TO HAVE OUTCOMES AT LEAST AS 1212 00:46:18,412 --> 00:46:22,216 GOOD OF LUNG TRANSPLANTATION. 1213 00:46:22,216 --> 00:46:23,250 AT WHAT POINT WOULD THE FAMILY 1214 00:46:23,250 --> 00:46:24,285 CHOOSE. 1215 00:46:24,285 --> 00:46:26,620 THE IRBROVAL IS IMPORTANT TO 1216 00:46:26,620 --> 00:46:28,389 CONSIDER AS THESE INFANTS WILL 1217 00:46:28,389 --> 00:46:30,691 BE AT LIKELY MULTIPLE CENTERS 1218 00:46:30,691 --> 00:46:31,759 THROUGHOUT THE UNITED STATES AND 1219 00:46:31,759 --> 00:46:33,460 HOW DO WE MEASURE CLINICAL 1220 00:46:33,460 --> 00:46:33,794 RESPONSE. 1221 00:46:33,794 --> 00:46:36,230 WE HAVE PF Ts FOR OLDER 1222 00:46:36,230 --> 00:46:38,199 CHILDREN, FOR INFANTS HAVE 1223 00:46:38,199 --> 00:46:40,000 FARE MEASURES TO MEASURE 1224 00:46:40,000 --> 00:46:41,869 CLINICAL RESPONSE. 1225 00:46:41,869 --> 00:46:47,274 THIS WOULD INCLUDE OXYGEN ATION 1226 00:46:47,274 --> 00:46:49,076 AND IMAGING FINDINGS THAT MAY 1227 00:46:49,076 --> 00:46:51,579 CHANGE. 1228 00:46:51,579 --> 00:46:54,215 I KNOW SEVERAL OTHER SPEAKERS 1229 00:46:54,215 --> 00:46:57,051 HAVE TALKED ABOUT N-OF-1 1230 00:46:57,051 --> 00:46:57,651 STUDIES. 1231 00:46:57,651 --> 00:47:00,688 THE RARENESS OF THESE DISORDERS 1232 00:47:00,688 --> 00:47:02,556 PROVIDES UNIQUE CHALLENGES FOR 1233 00:47:02,556 --> 00:47:04,124 TRIAL DESIGN. 1234 00:47:04,124 --> 00:47:05,092 THESE ARE IMPORTANT 1235 00:47:05,092 --> 00:47:05,726 CONSIDERATIONS. 1236 00:47:05,726 --> 00:47:08,062 I WILL HIGHLIGHT THIS ARTICLE 1237 00:47:08,062 --> 00:47:11,732 THAT WAS RECENTLY PUBLISHED WITH 1238 00:47:11,732 --> 00:47:16,937 A NICE APPROACH TO N-OF-1 1239 00:47:16,937 --> 00:47:17,504 TRIALS. 1240 00:47:17,504 --> 00:47:17,871 CONSIDERATIONS. 1241 00:47:17,871 --> 00:47:19,974 WE NEED INCREASED PROVIDER 1242 00:47:19,974 --> 00:47:23,510 EDUCATION FOR EARLIER IDENT 1243 00:47:23,510 --> 00:47:26,780 IDENTIFICATION AND INCREASED 1244 00:47:26,780 --> 00:47:31,151 ACCESSIBILITY TO WGS IN THE NICU 1245 00:47:31,151 --> 00:47:35,589 AND PEDIATRIC PULMONARY CLINICS. 1246 00:47:35,589 --> 00:47:38,359 MY RECOMMENDATION IS THEY ARE 1247 00:47:38,359 --> 00:47:41,262 TRANSPORTED TO CENTERS WHO HAVE 1248 00:47:41,262 --> 00:47:51,705 EXPERTISE WITH CHILD AND 1249 00:47:52,139 --> 00:47:56,644 TRANSPORT TEAMS. 1250 00:47:56,644 --> 00:48:00,014 I WANT TO ALSO SAY IDENTIFYING 1251 00:48:00,014 --> 00:48:01,015 INVESTIGATIONAL THERAPIES ONCE 1252 00:48:01,015 --> 00:48:01,282 AVAILABLE. 1253 00:48:01,282 --> 00:48:01,649 NEXT SLIDE. 1254 00:48:01,649 --> 00:48:03,617 AND THEN I THINK IT IS 1255 00:48:03,617 --> 00:48:04,952 INCREDIBLY IMPORTANT TO ENGAGE 1256 00:48:04,952 --> 00:48:07,054 THE PARENTS OF AFFECTED INFANTS 1257 00:48:07,054 --> 00:48:09,590 AND CHILDREN AND CERTAINLY THE 1258 00:48:09,590 --> 00:48:10,758 CHILD FOUNS HAS HELPED WITH 1259 00:48:10,758 --> 00:48:11,191 THIS. 1260 00:48:11,191 --> 00:48:12,793 I THINK WE NEED TO TALK TO THE 1261 00:48:12,793 --> 00:48:16,497 PARENTS OF INFANTS WHO WERE E 1262 00:48:16,497 --> 00:48:18,032 LANTED VERSUS FAMILIES WHO 1263 00:48:18,032 --> 00:48:18,832 PURSUED COMFORT CARE. 1264 00:48:18,832 --> 00:48:21,769 IN TALKING WITH FAMILIES ABOUT 1265 00:48:21,769 --> 00:48:23,037 TRANSPLANTATION ONCE THEY 1266 00:48:23,037 --> 00:48:25,773 HEAR ALL OF THE PROCEDURES AND 1267 00:48:25,773 --> 00:48:28,108 MAINTENANCE THAT IS INVOLVED AND 1268 00:48:28,108 --> 00:48:30,411 THE FIVE-YEAR SURVIVAL RATES, 1269 00:48:30,411 --> 00:48:32,780 HALF OF FAMILIES DO NOT A WANT 1270 00:48:32,780 --> 00:48:33,981 TO PURSUE TRANSPLANTATION FOR 1271 00:48:33,981 --> 00:48:35,049 THEIR CHILD. 1272 00:48:35,049 --> 00:48:37,751 ARE THESE FAMILIES WHO MAY 1273 00:48:37,751 --> 00:48:39,353 PURSUE INVESTIGATIONAL THERAPY? 1274 00:48:39,353 --> 00:48:41,822 WHAT ARE ETHICS AND CONSENT 1275 00:48:41,822 --> 00:48:44,058 ISSUES INVOLVED IN THAT PROCESOC 1276 00:48:44,058 --> 00:48:46,827 CERTAINLY IRB APPROVAL TAKES A 1277 00:48:46,827 --> 00:48:47,528 WHILE. 1278 00:48:47,528 --> 00:48:49,096 REG CENTERS 1279 00:48:49,096 --> 00:48:51,899 ALREADY READY TO HAVE AN 1280 00:48:51,899 --> 00:48:52,966 INVESTIGATIONAL THERAPY APPROVED 1281 00:48:52,966 --> 00:48:55,069 THROUGH THEIR IRB SO ONCE THESE 1282 00:48:55,069 --> 00:48:57,304 INFANTS ARE IDENTIFIED AND 1283 00:48:57,304 --> 00:48:59,340 ARRI AT CENTERS COULD 1284 00:48:59,340 --> 00:49:00,808 RECEIVE EXPEDITIOUS TREATMENT. 1285 00:49:00,808 --> 00:49:02,643 WE NEED TO THINK ABOUT CLINICAL 1286 00:49:02,643 --> 00:49:02,910 RESPONSE. 1287 00:49:02,910 --> 00:49:05,979 AS I MENTIONED, INFANTS, WE HAVE 1288 00:49:05,979 --> 00:49:08,282 LIMITED MEASURES OTHER THAN 1289 00:49:08,282 --> 00:49:10,551 VENTILATION, OXYGENATION STATUS. 1290 00:49:10,551 --> 00:49:16,090 WE NEED TO FOCUS ON BIOMARKERS. 1291 00:49:16,090 --> 00:49:18,325 MAYBE CHANGES IN IMAGING CAN BE 1292 00:49:18,325 --> 00:49:18,926 AN END POINT. 1293 00:49:18,926 --> 00:49:22,463 NEXT SLIDE. 1294 00:49:22,463 --> 00:49:24,531 WITH THAT I JUST WANT TO THANK 1295 00:49:24,531 --> 00:49:26,367 OUR TRANSPLANT TEAM AT ST. LOUIS 1296 00:49:26,367 --> 00:49:27,568 CHILDREN'S HOSPITAL. 1297 00:49:27,568 --> 00:49:31,171 OUR RESEARCH TEAM AND 1298 00:49:31,171 --> 00:49:35,376 COLLABORATORS ACROSS THE 1299 00:49:35,376 --> 00:49:35,609 COUNTRY. 1300 00:49:35,609 --> 00:49:35,943 THANK YOU. 1301 00:49:35,943 --> 00:49:37,778 >> THANK YOU SO MUCH, DR. 1302 00:49:37,778 --> 00:49:38,212 WAMBACH. 1303 00:49:38,212 --> 00:49:40,614 THAT WAS GREAT AND CERTAINLY 1304 00:49:40,614 --> 00:49:42,750 QUITE A TO-DO LIST, REALLY, IN 1305 00:49:42,750 --> 00:49:44,017 TERMS OF CONSIDERATIONS AND 1306 00:49:44,017 --> 00:49:45,619 THINGS TO TRY TO IMPROVE THIS 1307 00:49:45,619 --> 00:49:47,121 PROCESS. 1308 00:49:47,121 --> 00:49:50,591 SO WE'LL MOVE ON TO DORIS 1309 00:49:50,591 --> 00:49:52,226 SANCHEZ, OUR NEXT SPEAKER. 1310 00:49:52,226 --> 00:49:53,527 AND SO DORIS GO AHEAD AND TAKE 1311 00:49:53,527 --> 00:49:55,696 IT AWAY. 1312 00:49:55,696 --> 00:49:56,797 >> I'M ON MUTE. 1313 00:49:56,797 --> 00:49:58,132 I'M SORRY. 1314 00:49:58,132 --> 00:49:58,899 HELLO, EVERYONE. 1315 00:49:58,899 --> 00:50:00,334 MOST OF WHAT I'M COVERING TODAY 1316 00:50:00,334 --> 00:50:02,803 HAS BEEN PREN ONE FORM 1317 00:50:02,803 --> 00:50:04,471 OR ANOTHER DURING THIS WORKSHOP, 1318 00:50:04,471 --> 00:50:06,140 BUT IN GREAT DETAIL AS 1319 00:50:06,140 --> 00:50:08,175 INDIVIDUAL FOCUSED TOPICS. 1320 00:50:08,175 --> 00:50:09,243 THE REALITY IS OPTIMAL 1321 00:50:09,243 --> 00:50:11,311 EXECUTION OF CLINICAL TRIALSNI 1322 00:50:11,311 --> 00:50:13,547 NOT DONE IN A VACUUM, BUTCUER 1323 00:50:13,547 --> 00:50:15,783 A NUMBER OF BROADER ACTIVITIES 1324 00:50:15,783 --> 00:50:17,684 OCCUR TO INFORM A SOLL LIED 1325 00:50:17,684 --> 00:50:20,788 TRIAL DESIGN AND PROACTIVE TRIAL 1326 00:50:20,788 --> 00:50:21,555 EXECUTION. 1327 00:50:21,555 --> 00:50:23,791 I WILL BRING MANY O THE FOCUSED 1328 00:50:23,791 --> 00:50:25,025 TOPICS THAT HAVE BEEN D, 1329 00:50:25,025 --> 00:50:27,261 NOT IN DETAIL, THAT HAS BEEN 1330 00:50:27,261 --> 00:50:29,596 DONE, BUT A BROADER PERSPECTIVE 1331 00:50:29,596 --> 00:50:30,831 IN THE CONTEXT OF THE 1332 00:50:30,831 --> 00:50:32,733 CONSIDERATIONS OF DESIGNING AND 1333 00:50:32,733 --> 00:50:35,836 EXECUTING CLINICAL TRIALS. 1334 00:50:35,836 --> 00:50:38,672 SO THIS TOPIC CAN GO MANY WAYS. 1335 00:50:38,672 --> 00:50:40,240 I'M GOING TO FOCUS ON THREE 1336 00:50:40,240 --> 00:50:42,476 PRACTICAL ONES THAT I OFTEN WORK 1337 00:50:42,476 --> 00:50:44,578 ON WITH MY CLIENTS WORKING ON 1338 00:50:44,578 --> 00:50:47,214 RARE DISEASE IN ONCOLOGY, RARE 1339 00:50:47,214 --> 00:50:47,614 INDICATIONS. 1340 00:50:47,614 --> 00:50:49,149 THE FIRST ONE I'M GOING TO FOCUS 1341 00:50:49,149 --> 00:50:51,151 ON IS THE CLINICAL DEVELOPMENT 1342 00:50:51,151 --> 00:50:53,821 STRATEGYELEL WHICH IS BASICALLYR 1343 00:50:53,821 --> 00:50:56,757 BRAINSTORMING, RESEARCHING AND 1344 00:50:56,757 --> 00:50:57,724 SOLIDIFYING THOUGHTS TO BUILD 1345 00:50:57,724 --> 00:50:58,459 OVERALL STRATEGY. 1346 00:50:58,459 --> 00:51:00,461 THE NEXT IS CONSIDERATIONS THAT 1347 00:51:00,461 --> 00:51:02,796 HELP DEVELOPING A SOLID 1348 00:51:02,796 --> 00:51:04,331 CLINICAL TRIAL DESIGN AND THE 1349 00:51:04,331 --> 00:51:05,666 LAST IS CLINICAL TRIAL 1350 00:51:05,666 --> 00:51:05,933 EXECUTION. 1351 00:51:05,933 --> 00:51:08,802 NEXT SLIEX 1352 00:51:08,802 --> 00:51:10,637 SO YOUR CLINICAL DEVELOPMENT 1353 00:51:10,637 --> 00:51:10,904 STRATEGY. 1354 00:51:10,904 --> 00:51:13,307 I KIND OF PUT THAT IN THE 1355 00:51:13,307 --> 00:51:14,541 FOREFRONT BECAUSE THAT IS WHERE 1356 00:51:14,541 --> 00:51:16,877 YOU ARE ABLE TO DEFINE YOUR 1357 00:51:16,877 --> 00:51:18,245 OVERALL GOALS AND STRATEGY FOR A 1358 00:51:18,245 --> 00:51:18,712 PROGRAM. 1359 00:51:18,712 --> 00:51:20,314 THAT IS REALLY CRITICAL. 1360 00:51:20,314 --> 00:51:21,748 KIND OF LIKE IF YOU WANT TO 1361 00:51:21,748 --> 00:51:22,850 BUILD A HOUSE. 1362 00:51:22,850 --> 00:51:24,184 THIS IS WHERE YOU DETERMINE 1363 00:51:24,184 --> 00:51:25,986 WHERE YOU WANT TO BUILD, WHAT IS 1364 00:51:25,986 --> 00:51:27,621 YOUR BUDGET, WHAT TYPE OF HOUSE 1365 00:51:27,621 --> 00:51:29,356 DO YOU WANT? 1366 00:51:29,356 --> 00:51:31,091 WHAT ARE YOUR DEAL BREAKERS IN 1367 00:51:31,091 --> 00:51:33,927 WHAT BUILDER ARE YOU GOING TO 1368 00:51:33,927 --> 00:51:34,094 USE? 1369 00:51:34,094 --> 00:51:35,496 AND YOU START LOOKING AT YOUR 1370 00:51:35,496 --> 00:51:37,164 FLOOR PLAN. 1371 00:51:37,164 --> 00:51:38,599 YOU HAVE A PRELIMINARY PLAN THAT 1372 00:51:38,599 --> 00:51:40,033 GUIDES YOUR PROCESS FROM 1373 00:51:40,033 --> 00:51:41,468 BEGINNING TO END. 1374 00:51:41,468 --> 00:51:43,237 THINKING ABOUT BUILDING A 1375 00:51:43,237 --> 00:51:44,838 CLINICALMENT STRATEGY I S 1376 00:51:44,838 --> 00:51:46,440 BRAINSTORM THREE AREAS AND IT 1377 00:51:46,440 --> 00:51:47,274 WORKS OUT. 1378 00:51:47,274 --> 00:51:49,810 WHAT IS YOUR REGULATORY 1379 00:51:49,810 --> 00:51:52,880 STRATEGY, ASSET STRATEGY AND 1380 00:51:52,880 --> 00:51:55,983 OVERALL GOALS AND ASSETS PROOF 1381 00:51:55,983 --> 00:51:58,785 OF MECHANISM, PROOF OF PRINCIPLE 1382 00:51:58,785 --> 00:52:01,555 AND PROOF OF CONCEPT. 1383 00:52:01,555 --> 00:52:03,524 THE REASON I PRIORITIZE 1384 00:52:03,524 --> 00:52:04,324 REGULATORY STRATEGY IS NOT 1385 00:52:04,324 --> 00:52:05,659 BECAUSE IT IS MORE IMPORTANT 1386 00:52:05,659 --> 00:52:06,927 THAN ALL THE OTHER ELEMENTS 1387 00:52:06,927 --> 00:52:08,929 BECAUSE I THINK IT IS ONE OF THE 1388 00:52:08,929 --> 00:52:10,898 CORE ELEMENTS THAT GUIDES AND 1389 00:52:10,898 --> 00:52:11,899 INFORMS OVERALL PLAN. 1390 00:52:11,899 --> 00:52:14,067 THIS IS WHERE YOU FIGURE OUT 1391 00:52:14,067 --> 00:52:15,168 UNITIES FOR APPROVART 1392 00:52:15,168 --> 00:52:15,502 PATHWAYS. 1393 00:52:15,502 --> 00:52:17,971 YOU DEFINE WHAT IS YOUR IDEAL 1394 00:52:17,971 --> 00:52:20,073 LABEL AND WORK BACKWARDS AND 1395 00:52:20,073 --> 00:52:21,441 THAT WILL OFTEN DEFINE YOUR 1396 00:52:21,441 --> 00:52:22,709 PHASE 1 TRIAL. 1397 00:52:22,709 --> 00:52:23,644 WHAT ARE THE MINIMUM 1398 00:52:23,644 --> 00:52:26,747 REQUIREMENTS TO GET INTO THE 1399 00:52:26,747 --> 00:52:27,147 CLINIC? 1400 00:52:27,147 --> 00:52:29,316 THAT WILL DEFINITELY DEFINE THE 1401 00:52:29,316 --> 00:52:30,250 EXPERIMENTS YOU ARE GOING TO RUN 1402 00:52:30,250 --> 00:52:31,318 IN SOMEONE. 1403 00:52:31,318 --> 00:52:33,453 BUILDING A REGULATORY ROAD MAP 1404 00:52:33,453 --> 00:52:35,722 IS A GREAT TOOL THAT WE USE TO 1405 00:52:35,722 --> 00:52:37,190 INFORM THE CLINICAL DEVELOPMENT 1406 00:52:37,190 --> 00:52:39,059 STRATEGY, BUT ALSO ALLF YOUR 1407 00:52:39,059 --> 00:52:43,196 OTHER FUNCTIONAL STRATEGIES, 1408 00:52:43,196 --> 00:52:43,564 CMC, ET CETERA. 1409 00:52:43,564 --> 00:52:46,199 SO THEN ONCE YOU HAVE YOUR 1410 00:52:46,199 --> 00:52:47,200 REGULATORY STRATEGY IN PARALLEL 1411 00:52:47,200 --> 00:52:49,736 YOU ARE ACTUALLY WORKING ON YOUR 1412 00:52:49,736 --> 00:52:50,871 ASSET STRATEGY. 1413 00:52:50,871 --> 00:52:53,640 THE REASON I ISOLATE THE 1414 00:52:53,640 --> 00:52:55,809 REGULATORY STRATEGY IS BECAUSE 1415 00:52:55,809 --> 00:52:57,377 REGULATORY IS ONE KEY 1416 00:52:57,377 --> 00:52:59,513 STAKEHOLDER IN EACH OF THESE 1417 00:52:59,513 --> 00:52:59,913 EXERCISES. 1418 00:52:59,913 --> 00:53:03,584 SO AGAIN, NOT TO SAY THESE 1419 00:53:03,584 --> 00:53:05,419 EXERCISES ARE NOT IMPORTANT.OT 1420 00:53:05,419 --> 00:53:07,421 SO THEOT KEY ACTIVITY 1421 00:53:07,421 --> 00:53:09,556 BUILDING THE ASSET SPECIFIC 1422 00:53:09,556 --> 00:53:09,890 STRATEGY. 1423 00:53:09,890 --> 00:53:12,759 BUILDING YOUR ASSET STRATEGY 1424 00:53:12,759 --> 00:53:14,494 IS WHERE ONE RESEARCHS, 1425 00:53:14,494 --> 00:53:16,330 BRAINSTORMS AND STARTS TO PUT 1426 00:53:16,330 --> 00:53:17,297 PEN TO PAPER. 1427 00:53:17,297 --> 00:53:19,266 I DESCRIBE IT TO MANY OF MY 1428 00:53:19,266 --> 00:53:21,201 CLIENTS AS PUTTING ALL YOUR 1429 00:53:21,201 --> 00:53:24,404 STICKY NOTES ON THE WALL, 1430 00:53:24,404 --> 00:53:25,639 PRIORITIZING THE INDICATIONS YOU 1431 00:53:25,639 --> 00:53:28,709 WANT TO GO INTO OVERALL AND IN 1432 00:53:28,709 --> 00:53:29,776 YOUR FIRST HUMAN TRIAL. 1433 00:53:29,776 --> 00:53:31,144 ALL OF YOUR OPPORTUNITIES YOU 1434 00:53:31,144 --> 00:53:33,447 CAN DEVELOP IN YOUR PROGRAM 1435 00:53:33,447 --> 00:53:34,915 VERSUS LIKE YOUR FLAGSHIP 1436 00:53:34,915 --> 00:53:35,882 INDICATION THAT YOU ARE GOING TO 1437 00:53:35,882 --> 00:53:38,385 START WITH OR THAT YOU WANT TO 1438 00:53:38,385 --> 00:53:40,854 GO FIRST FOR APPROVAL. 1439 00:53:40,854 --> 00:53:42,055 BUILDING ANEGRATED 1440 00:53:42,055 --> 00:53:42,756 DEVELOLAN IS ANOTHER 1441 00:53:42,756 --> 00:53:44,424 HA CR 1442 00:53:44,424 --> 00:53:47,461 AND THAT FACTORS IN CROSS 1443 00:53:47,461 --> 00:53:51,131 FUNCTIONALLY YOUR CMC, 1444 00:53:51,131 --> 00:53:51,632 PRECLINICAL MARKETING 1445 00:53:51,632 --> 00:53:53,233 COMMERCIAL, ALL OF THESE 1446 00:53:53,233 --> 00:53:54,034 ELEMENTS THAT BUILD YOUR 1447 00:53:54,034 --> 00:53:55,302 DEVELOPMENT PLAN. 1448 00:53:55,302 --> 00:53:57,738 I LOOK AT IT MORE AS INTEGRATED 1449 00:53:57,738 --> 00:53:59,406 PLAN AND NOT JUST A CLINICAL 1450 00:53:59,406 --> 00:54:00,007 PLAN. 1451 00:54:00,007 --> 00:54:02,876 THAT HELPS FOR ALL OF THOSE 1452 00:54:02,876 --> 00:54:04,711 AREAS TO ALIGN CROSS 1453 00:54:04,711 --> 00:54:07,614 FUNCTIONALLY SO THERE IS NOT 1454 00:54:07,614 --> 00:54:08,181 MISCOMMUNICATION OR 1455 00:54:08,181 --> 00:54:08,548 MISINFORMATION. 1456 00:54:08,548 --> 00:54:10,817 EVEN IF IT IS JUST A SIMPLE 1457 00:54:10,817 --> 00:54:12,152 POWER POINT SET OF SLIDES. 1458 00:54:12,152 --> 00:54:14,655 DEVELOPING AN INTEGRATED TARGET 1459 00:54:14,655 --> 00:54:17,391 PROFILE IS ANOTHER PROCESS. 1460 00:54:17,391 --> 00:54:20,160 IF YOU NOTICE I'M USING THE WORD 1461 00:54:20,160 --> 00:54:20,460 INTEGRATED. 1462 00:54:20,460 --> 00:54:22,195 THAT IS WHERE YOU LOOK AT ALL OF 1463 00:54:22,195 --> 00:54:24,564 THOSE ELEMENTS LIKE CMC, 1464 00:54:24,564 --> 00:54:26,466 CLINICAL, PARAMETERS THAT FLAG 1465 00:54:26,466 --> 00:54:28,268 WHAT LOOKS GOOD, WHAT LOOKS BAD 1466 00:54:28,268 --> 00:54:29,670 AND WHAT IS GOING TO MAKE THINGS 1467 00:54:29,670 --> 00:54:31,204 SO AMAZING? 1468 00:54:31,204 --> 00:54:34,574 AND THEN AFTER THE T PP, OFTEN 1469 00:54:34,574 --> 00:54:36,643 WILL ENCOURAGE CLIENTS 1470 00:54:36,643 --> 00:54:39,579 YOUR TOP ONE TONE THREE THAT WE 1471 00:54:39,579 --> 00:54:41,548 CALL IT VALUE CREATION PLAN OR 1472 00:54:41,548 --> 00:54:43,617 MANY OTHER THINGS YOU CALL IT. 1473 00:54:43,617 --> 00:54:47,454 IS YOUR GO, NO GO CRITERIA. 1474 00:54:47,454 --> 00:54:49,423 THIS IS YOUR BIG -- COULD 1475 00:54:49,423 --> 00:54:51,158 TERMINATE YOUR PROGRAM OR BE 1476 00:54:51,158 --> 00:54:54,494 WHERE YOU DROP EVERYTHING AND GO 1477 00:54:54,494 --> 00:54:55,462 FAST TO APPROVAL. 1478 00:54:55,462 --> 00:55:02,002 SO NEXT SLIDE. 1479 00:55:02,002 --> 00:55:03,870 SO THEN I OFTEN HAVE CLIENTS SAY 1480 00:55:03,870 --> 00:55:06,973 WHY ARE WE THINKING OF PROOF OF 1481 00:55:06,973 --> 00:55:08,542 PRINCIPLE, PROOF OF MECHANISM 1482 00:55:08,542 --> 00:55:09,943 AND PROOF OF CONCEPT? 1483 00:55:09,943 --> 00:55:12,179 THE REASON ISNFTER YOU HAVE AN 1484 00:55:12,179 --> 00:55:14,614 IDEA OF REGULATORY PATH AND 1485 00:55:14,614 --> 00:55:18,285 OVERALL ASSET STRATEGIES THAT 1486 00:55:18,285 --> 00:55:20,554 TELLS YOU INDIC AND 1487 00:55:20,554 --> 00:55:22,189 PROPERTIES OF YOUR PRODUCT AND 1488 00:55:22,189 --> 00:55:24,458 HOW YOU WANT TO GET THROUGH YOUR 1489 00:55:24,458 --> 00:55:26,993 TRIAL AND APPROVAL. 1490 00:55:26,993 --> 00:55:30,697 ONE BIG EXERCISE SO TO ACHIEVE 1491 00:55:30,697 --> 00:55:33,066 DEMONSTRATING YOUR PROOF OF 1492 00:55:33,066 --> 00:55:37,404 PRINCIPAL, PROOF OF MECHANISM 1493 00:55:37,404 --> 00:55:39,506 AND PROOF OF CONCEPT. 1494 00:55:39,506 --> 00:55:41,141 THAT IT SHOULD BE DOING WHAT YOU 1495 00:55:41,141 --> 00:55:42,075 THINK IT SHOULD BE DOING. 1496 00:55:42,075 --> 00:55:43,744 IF NOT, WHAT IS HAPPENING. 1497 00:55:43,744 --> 00:55:45,879 IT HELPS YOU TO START TO THINK 1498 00:55:45,879 --> 00:55:47,380 ABOUT HOW TO MEASURE THESE 1499 00:55:47,380 --> 00:55:49,316 ELEMENTS BECAUSE OFTENTIMES WE 1500 00:55:49,316 --> 00:55:51,051 NEED LONG LEAD TIMES TO DEVELOP 1501 00:55:51,051 --> 00:55:55,322 CLINICAL ASSAYS. 1502 00:55:55,322 --> 00:55:58,725 DEFINES POM, POP AND POC WILL 1503 00:55:58,725 --> 00:55:59,793 DEFINE STRATEGIES AND SOME OF 1504 00:55:59,793 --> 00:56:00,994 YOUR END POINTS. 1505 00:56:00,994 --> 00:56:04,064 NEXT SLIDE. 1506 00:56:04,064 --> 00:56:05,198 ALL RIGHT. 1507 00:56:05,198 --> 00:56:08,068 CLINICAL TRIAL CONSIDERATIONS. 1508 00:56:08,068 --> 00:56:10,904 ONCE YOU HAVE AT LEAST A FIRST 1509 00:56:10,904 --> 00:56:15,709 PASS OF AN OVERALL DEVELOPMENTOA 1510 00:56:15,709 --> 00:56:20,413 DEEPER DIVE INTO YOUR TRIAL 1511 00:56:20,413 --> 00:56:22,115 DESIGN IS MUCH EASIER AND HERE 1512 00:56:22,115 --> 00:56:23,416 IS WHY. 1513 00:56:23,416 --> 00:56:24,985 I WAS TALKING ABOUT THE PROCESS 1514 00:56:24,985 --> 00:56:26,419 OF BUILDING A HOUSE. 1515 00:56:26,419 --> 00:56:27,988 BY THIS POINT YOU BOUGHT A PIECE 1516 00:56:27,988 --> 00:56:30,090 OF LAND, HAVE AN IDEA OF WHAT 1517 00:56:30,090 --> 00:56:32,125 YOU CAN AND CANNOT BUILD, THE 1518 00:56:32,125 --> 00:56:34,728 NUANCES OF THE LAND SURVEY AND 1519 00:56:34,728 --> 00:56:36,029 WRITTEN DOWN YOUR WISH LIST, THE 1520 00:56:36,029 --> 00:56:37,631 F MAYND HAVE 1521 00:56:37,631 --> 00:56:38,999 PICKED YOU FLOOR PLAN. 1522 00:56:38,999 --> 00:56:40,867 NOW THAT YOU HAVE ALL THOSE 1523 00:56:40,867 --> 00:56:43,770 THINGS IN MIND IT IS TIME TO 1524 00:56:43,770 --> 00:56:44,704 CUSTOMIZE YOUR BLUEPRINT. 1525 00:56:44,704 --> 00:56:47,107 THAT IS WHAT I CONSIDER YOUR 1526 00:56:47,107 --> 00:56:48,375 CLINICAL TRIAL DESIGN 1527 00:56:48,375 --> 00:56:49,309 CONSIDERATIONS WHERE YOU GET 1528 00:56:49,309 --> 00:56:51,211 INTO THE NITTY-GRITTY. 1529 00:56:51,211 --> 00:56:52,712 I CALL IT THE FUN PART. 1530 00:56:52,712 --> 00:56:56,316 NEXT SLIDE. 1531 00:56:56,316 --> 00:56:58,318 SO SAME THING AS A CLINICAL 1532 00:56:58,318 --> 00:56:59,820 TRIAL AND THAT EXAMPLE OF THE 1533 00:56:59,820 --> 00:57:00,687 HOUSE BUILDING. 1534 00:57:00,687 --> 00:57:02,689 I LOOK AT FOUR BIG BUCKETS TO 1535 00:57:02,689 --> 00:57:05,025 DEFINE AND GUIDE A CLINICAL 1536 00:57:05,025 --> 00:57:06,193 TRIAL DESIGN. 1537 00:57:06,193 --> 00:57:07,894 THERE ARE MANY MORE WAYS TO LOOK 1538 00:57:07,894 --> 00:57:08,728 AT IT. 1539 00:57:08,728 --> 00:57:12,132 THE VERY FIRST THING IS YOUR 1540 00:57:12,132 --> 00:57:13,800 PATIENT CENTERICITY. 1541 00:57:13,800 --> 00:57:15,435 WHAT IS HAPPENING IN REAL LIFE? 1542 00:57:15,435 --> 00:57:18,638 WHO ARE THE PATIENTS? 1543 00:57:18,638 --> 00:57:19,906 WHAT IS THEIR JOURNEY? 1544 00:57:19,906 --> 00:57:22,642 HOW ARE THEY TREATED? 1545 00:57:22,642 --> 00:57:24,911 HOW DOES YOUR ASSAY FIT THIS 1546 00:57:24,911 --> 00:57:25,178 PARADIGM? 1547 00:57:25,178 --> 00:57:26,980 WHO IS THE KEY DOCTOR OR TEAM 1548 00:57:26,980 --> 00:57:28,315 EA THE PATIENT? 1549 00:57:28,315 --> 00:57:30,917 IDEAL SITE AND 1550 00:57:30,917 --> 00:57:32,485 SETTING YOU NEED TO TRY TO BAKE 1551 00:57:32,485 --> 00:57:34,054 INTO YOUR CLINICAL TRIAL. 1552 00:57:34,054 --> 00:57:36,056 SO MAPPING OUT REAL LIFE 1553 00:57:36,056 --> 00:57:37,157 SOMETIMES WE CAN'T DO EVERYTHING 1554 00:57:37,157 --> 00:57:39,326 IN A CLINICAL TRIAL, BUT I 1555 00:57:39,326 --> 00:57:41,094 ALWAYS LAY THATWA FIRST AND I 1556 00:57:41,094 --> 00:57:43,563 GUIDE TO TRY TO 1557 00:57:43,563 --> 00:57:45,699 MODEL IT AS CLOSELY AS POSSIBLE 1558 00:57:45,699 --> 00:57:47,067 IN MY CLINICAL TRIAL. 1559 00:57:47,067 --> 00:57:49,102 AND THE NEXT IS YOUR TRIAL 1560 00:57:49,102 --> 00:57:49,936 DESIGN. 1561 00:57:49,936 --> 00:57:53,406 IT IS BUILDING YOUR PROTOCOL 1562 00:57:53,406 --> 00:57:53,907 SHELL. 1563 00:57:53,907 --> 00:58:01,581 MAPPING OUT YOUR ASSUMPASSUMPTI 1564 00:58:01,581 --> 00:58:05,652 POTENTIAL DESIGN,, WHAT ARE YOU 1565 00:58:05,652 --> 00:58:06,953 IAL OBJECTIVES AND 1566 00:58:06,953 --> 00:58:08,154 REMEMBER IN THE PREVIOUS 1567 00:58:08,154 --> 00:58:09,923 EXERCISES YOU HAVE ANSWERS LIKE 1568 00:58:09,923 --> 00:58:11,958 HOW WE MEASURE PROOF OF CONCEPT, 1569 00:58:11,958 --> 00:58:13,660 PROOF OF PRINCIPAL AND ALL OF 1570 00:58:13,660 --> 00:58:14,194 THAT. 1571 00:58:14,194 --> 00:58:15,295 ANOTHER ONE IS OPERATIONAL 1572 00:58:15,295 --> 00:58:15,562 PLANNING. 1573 00:58:15,562 --> 00:58:17,297 I MEAN, YOU NEED TO MAKE SURE 1574 00:58:17,297 --> 00:58:19,232 YOU KNOW WHAT IT IS GOING TO 1575 00:58:19,232 --> 00:58:21,768 COST, HOW LONG IT IS GOING TO 1576 00:58:21,768 --> 00:58:23,336 TAKE AND AGAINST HOW YOU THINK 1577 00:58:23,336 --> 00:58:24,638 IT IS GOING TO ENROLL. 1578 00:58:24,638 --> 00:58:26,306 WHAT RESOURCES YOU NEED TO GET 1579 00:58:26,306 --> 00:58:27,207 IT DONE. 1580 00:58:27,207 --> 00:58:29,376 WHO ARE YOUR VENDORS? 1581 00:58:29,376 --> 00:58:31,177 WHAT ARE YOUR RISKS? 1582 00:58:31,177 --> 00:58:32,979 BASICALLY HOW ARE YOU GOING TO 1583 00:58:32,979 --> 00:58:33,580 GET IT DONE? 1584 00:58:33,580 --> 00:58:35,415 DO YOU HAVE ALL THE RESOURCES TO 1585 00:58:35,415 --> 00:58:39,085 GET IT DONE? COME FROM A TRANS 1586 00:58:39,085 --> 00:58:40,954 MEDICINE BACKGROUND SO I AM A 1587 00:58:40,954 --> 00:58:43,290 LITTLE BIASED, BUT IT IS 1588 00:58:43,290 --> 00:58:45,425 ACTUALLY TRUE IF YOU BUILD A 1589 00:58:45,425 --> 00:58:48,561 CLINICAL TRANSLATIONAL PLAN 1590 00:58:48,561 --> 00:58:50,297 OTHER NAMES PEOPLE GIVE TO THIS, 1591 00:58:50,297 --> 00:58:53,767 A SIMPLE TABLE TO ITEMIZE YOUR 1592 00:58:53,767 --> 00:58:56,202 KEY QUESTIONS, WHAT ASSAYS CAN 1593 00:58:56,202 --> 00:58:58,038 ANSWER THOSE QUESTIONS, WHAT END 1594 00:58:58,038 --> 00:59:01,441 POINT DOES IT FEED INTO? 1595 00:59:01,441 --> 00:59:05,111 IS IT EXPLORATORY, IS IT 1596 00:59:05,111 --> 00:59:05,378 CRITICAL? 1597 00:59:05,378 --> 00:59:06,980 TALASIC TABLE 1598 00:59:06,980 --> 00:59:08,915 FRAMEW TAM CRITICAL BECAUSE IT 1599 00:59:08,915 --> 00:59:10,350 TIES INTO ALL OF THESE OTHER 1600 00:59:10,350 --> 00:59:11,451 PIECES AND YOU WILL SEE IN A 1601 00:59:11,451 --> 00:59:12,385 LATER SLIDE. 1602 00:59:12,385 --> 00:59:16,489 NEXT SLIDE. 1603 00:59:16,489 --> 00:59:18,558 SO THIS IS ACTUALLY, I'M NOT 1604 00:59:18,558 --> 00:59:19,826 GOING TO PRESENT THIS SLIDE, BUT 1605 00:59:19,826 --> 00:59:21,594 YOU WILL HAVE IT IN T I DECK. 1606 00:59:21,594 --> 00:59:29,102 THESE -- I'M PUTTING DOWN 1607 00:59:29,102 --> 00:59:31,004 QUESTIONS WHEN YOU ARE TRYING TO 1608 00:59:31,004 --> 00:59:33,239 BUILD AN EFFECTIVE BLUEPRINT 1609 00:59:33,239 --> 00:59:35,675 THERE ARE QUESTIONS YOU WANT TO 1610 00:59:35,675 --> 00:59:35,842 ASK. 1611 00:59:35,842 --> 00:59:37,944 TRIAL SPECIFIC QUESTIONS AND 1612 00:59:37,944 --> 00:59:39,079 BUSINESS QUESTIONS. 1613 00:59:39,079 --> 00:59:40,714 WE DO NEED TO ASK BUSINESS 1614 00:59:40,714 --> 00:59:42,048 QUESTIONS BECAUSE THERE ARE SOME 1615 00:59:42,048 --> 00:59:44,017 BUSINESS DRIVERS THAT MAY 1616 00:59:44,017 --> 00:59:49,622 INFLUENCE YOUR CLINICAL TRIAL 1617 00:59:49,622 --> 00:59:49,856 DESIGN. 1618 00:59:49,856 --> 00:59:53,626 FOR THE BETTER TO ENHANCE OUR 1619 00:59:53,626 --> 00:59:55,328 TRIAL DESIGN. 1620 00:59:55,328 --> 00:59:58,198 THE PURPOSE OF THIS SLIDE IS -- 1621 00:59:58,198 --> 00:59:59,399 SORRY ABOUT THAT. 1622 00:59:59,399 --> 01:00:03,370 NEXT SLIDE. 1623 01:00:03,370 --> 01:00:03,837 ALL RIGHT. 1624 01:00:03,837 --> 01:00:05,438 SO WE HAVE BEEN TALKING ABOUT 1625 01:00:05,438 --> 01:00:08,575 THE STRATEGY AND MAPPING OUT THE 1626 01:00:08,575 --> 01:00:10,276 SHELL AND OUTLINING THE PEOPLE, 1627 01:00:10,276 --> 01:00:13,279 THE PATIENTS AND THE POTENTIAL 1628 01:00:13,279 --> 01:00:13,513 MARKERS. 1629 01:00:13,513 --> 01:00:15,648 BUT ONE THING THAT IS CRITICAL 1630 01:00:15,648 --> 01:00:19,019 IS REALLY UNDERSTANDING WHAT 1631 01:00:19,019 --> 01:00:20,387 YOU'RE TRYING TO A TH INIS 1632 01:00:20,387 --> 01:00:22,489 PHASE 1 TRIAL, PHASE 2, PHASE 3, 1633 01:00:22,489 --> 01:00:23,623 PHASE 4. 1634 01:00:23,623 --> 01:00:25,291 IF YOU ARE JUST STARTING OUT IT 1635 01:00:25,291 --> 01:00:27,027 WOULD BE A PHASE 1 OR PHASE 1636 01:00:27,027 --> 01:00:27,627 COMBO 2. 1637 01:00:27,627 --> 01:00:29,929 THIS IS GOING TO BE REALLY 1638 01:00:29,929 --> 01:00:31,731 IMPORTANT TO UNDERSTAND. 1639 01:00:31,731 --> 01:00:32,966 WHAT ARE YOUR OPTIONS FOR 1640 01:00:32,966 --> 01:00:35,301 INSTANCE IN YOUR PHASE 1, A 1641 01:00:35,301 --> 01:00:37,871 HEALTHY NORMAL VOLUNTEER, 1642 01:00:37,871 --> 01:00:39,272 STRAIGHT INTO DISEASE PATIENTS? 1643 01:00:39,272 --> 01:00:41,241 WHAT IS THAT STRATEGY? 1644 01:00:41,241 --> 01:00:43,910 IS IT SINGLE ASCENDING, MULTS PL 1645 01:00:43,910 --> 01:00:45,912 ASCENDING OR DOSE LEVELS AND 1646 01:00:45,912 --> 01:00:47,614 DOSE ESCALATE AND DOSE FIND TO 1647 01:00:47,614 --> 01:00:49,282 FIND YOUR MAXIMUM TOLERATED 1648 01:00:49,282 --> 01:00:49,716 DOSE? 1649 01:00:49,716 --> 01:00:52,819 ARE YOU LOOKING AT DRUG 1650 01:00:52,819 --> 01:00:53,686 INTERACTION? 1651 01:00:53,686 --> 01:00:54,020 PK. 1652 01:00:54,020 --> 01:00:56,022 SO A LOT OF THESE THINGS WILL 1653 01:00:56,022 --> 01:00:57,724 HELP YOU KIND OF DEFINE THE 1654 01:00:57,724 --> 01:01:00,660 PHASE OF TRIAL YOU NEED TO FOCUS 1655 01:01:00,660 --> 01:01:00,960 ON. 1656 01:01:00,960 --> 01:01:02,328 I'M GOING TO IN ANOTHER SLIDE 1657 01:01:02,328 --> 01:01:04,764 TALK MORE ABOUT INNOVATING. 1658 01:01:04,764 --> 01:01:06,232 BUT AT LEAST HAVE AN IDEA OF 1659 01:01:06,232 --> 01:01:07,667 WHAT YOUR OPTIONS ARE BECAUSE A 1660 01:01:07,667 --> 01:01:09,736 LOT OF TIMES PEOPLE WILL THINK 1661 01:01:09,736 --> 01:01:12,172 PHASE 1, I CAN ONLY DO THIS. 1662 01:01:12,172 --> 01:01:15,175 KNOW WHAT YOUR OPTIONS ARE 1663 01:01:15,175 --> 01:01:17,010 WITHIN THAT PHASE 1, 2 AREA. 1664 01:01:17,010 --> 01:01:19,412 A PHASE 3, WHAT ARE THE CRITICAL 1665 01:01:19,412 --> 01:01:20,613 ELEMENTS OF THE PHASE 3 TO GET 1666 01:01:20,613 --> 01:01:22,215 TO APPROVAL AND WHAT ARE 1667 01:01:22,215 --> 01:01:23,516 REQUIREMENTS IF YOU ARE 1668 01:01:23,516 --> 01:01:24,684 DESIGNING A PHASE 4? 1669 01:01:24,684 --> 01:01:26,553 WHAT IS THE PURPOSE? 1670 01:01:26,553 --> 01:01:27,987 WHY ARE YOU EVEN DOING THIS 1671 01:01:27,987 --> 01:01:29,589 PHASE 4 AND ACTUALLY MAKING SURE 1672 01:01:29,589 --> 01:01:32,926 YOU ARE FOCUSED. 1673 01:01:32,926 --> 01:01:36,229 SO NEXT SLIDE. 1674 01:01:36,229 --> 01:01:36,596 ALL RIGHT. 1675 01:01:36,596 --> 01:01:38,231 SO INNOVATION. 1676 01:01:38,231 --> 01:01:39,966 NOW MORE THAN EVER WE WANT TO 1677 01:01:39,966 --> 01:01:42,836 GET PRODUCTSTRORO TO PATIENTS OH 1678 01:01:42,836 --> 01:01:46,072 UNMET NEED AS SAFELY, BUT AS 1679 01:01:46,072 --> 01:01:48,408 QUICKLY AS POSSIBLE AND ALSO AS 1680 01:01:48,408 --> 01:01:49,576 EFFECTIVELY AS POSSIBLE. 1681 01:01:49,576 --> 01:01:51,311 HERE ARE SOME CONSIDERATIONS WE 1682 01:01:51,311 --> 01:01:53,012 SHOULD BRAINSTORM TO DETERMINE 1683 01:01:53,012 --> 01:01:55,115 WAYS TO INNOVATE CLINICAL 1684 01:01:55,115 --> 01:01:55,348 TRIALS. 1685 01:01:55,348 --> 01:01:56,950 IS IT POSSIBLE TO ANSWER YOUR 1686 01:01:56,950 --> 01:01:59,252 PROOF OF CONCEPT, PROOF OF 1687 01:01:59,252 --> 01:02:01,221 MECHANISM AND PROOF OF PRINCIPAL 1688 01:02:01,221 --> 01:02:02,555 IN AN EARLY PHASE TRIAL? 1689 01:02:02,555 --> 01:02:03,556 THE ANSWER IS YES. 1690 01:02:03,556 --> 01:02:07,293 THE WAY WE DO THAT IS WE DESIGN 1691 01:02:07,293 --> 01:02:10,763 A PHASE 1, 2 TRIAL. 1692 01:02:10,763 --> 01:02:11,865 IS IT COMPLEX? 1693 01:02:11,865 --> 01:02:12,031 YES. 1694 01:02:12,031 --> 01:02:15,635 WE ARE DOING IT A LOT NOWADAYS 1695 01:02:15,635 --> 01:02:18,138 SO WE ARE COMPLETE WITH THE 1696 01:02:18,138 --> 01:02:20,773 FIRST TRIAL WE ARE AT PHASE 2 1697 01:02:20,773 --> 01:02:22,108 FIGURING OUT APPROVAL PATHWAY 1698 01:02:22,108 --> 01:02:23,243 WITH PHASE 3. 1699 01:02:23,243 --> 01:02:24,677 IT IS NOT IN THIS SLIDE, BUT ONE 1700 01:02:24,677 --> 01:02:26,679 OF THE THINGS THAT IS REALLY 1701 01:02:26,679 --> 01:02:29,649 CRITICAL, IF YOUR INDICATION IS 1702 01:02:29,649 --> 01:02:31,317 PRIMARILY OR IT IS ADULT AND A 1703 01:02:31,317 --> 01:02:33,786 HUGE PROPORTION O YOUNG 1704 01:02:33,786 --> 01:02:35,488 PATIENTS, ONE THING I OFTEN DO 1705 01:02:35,488 --> 01:02:37,257 IS FIGURE OUT HOW TO BAKE IN 1706 01:02:37,257 --> 01:02:39,859 YOUR YOUNGER POPULATIONS SOONER 1707 01:02:39,859 --> 01:02:40,994 VERSUS LATER. 1708 01:02:40,994 --> 01:02:43,730 THAT IS ANOTHER WAY TO INNOVATE. 1709 01:02:43,730 --> 01:02:46,099 THAT I'VE DONE WITH A LOT OF MY 1710 01:02:46,099 --> 01:02:48,501 CLIENTS LIKE A PHASE 1, 2 AND IN 1711 01:02:48,501 --> 01:02:50,136 YOUR PHASE 2, ONCE YOU 1712 01:02:50,136 --> 01:02:52,972 ESTABLISHED YOUR DOSE AND WITH 1713 01:02:52,972 --> 01:02:55,441 ADULTS MAYBE YOU EXPLO A 1714 01:02:55,441 --> 01:02:57,310 COHORT WITH SOME ADOLESCENT 1715 01:02:57,310 --> 01:02:58,411 PATIENTS IF YOU HAVE A CERTAIN 1716 01:02:58,411 --> 01:02:59,112 AMOUNT OF DATA. 1717 01:02:59,112 --> 01:03:01,281 WE HAVE GOTTEN THAT CLEARED WITH 1718 01:03:01,281 --> 01:03:02,615 THE FDA VERY RECENTLY. 1719 01:03:02,615 --> 01:03:05,385 SO THINKING OF TRIAL DESIGN 1720 01:03:05,385 --> 01:03:06,853 INNOVATION WITH IN MIND OF 1721 01:03:06,853 --> 01:03:10,056 GETTING THAT PRODUCT AS QUICKLY 1722 01:03:10,056 --> 01:03:12,058 AND AS SAFELY AND AS EFFECTIVELY 1723 01:03:12,058 --> 01:03:14,194 ON THE MARKET FOR THE PATIENTS 1724 01:03:14,194 --> 01:03:14,527 THAT NEED IT. 1725 01:03:14,527 --> 01:03:20,066 SO NEXT SLIDE. 1726 01:03:20,066 --> 01:03:21,968 SO THIS IS A SLIDE THAT IS A 1727 01:03:21,968 --> 01:03:22,502 RESOURCEFUL TOOL. 1728 01:03:22,502 --> 01:03:23,903 WE TALKED ABOUT EARLIER ONE OF 1729 01:03:23,903 --> 01:03:26,606 THE EXERCISES OF SKETCHING OUT 1730 01:03:26,606 --> 01:03:31,945 YOUR SIN -- SYNOPSIS OR 1731 01:03:31,945 --> 01:03:33,146 CLINICAL OUTLINE. 1732 01:03:33,146 --> 01:03:34,447 BASICALLY WHEN PCA THINK I'M 1733 01:03:34,447 --> 01:03:36,216 GOING TO DESIGN AN OUTLINE 1734 01:03:36,216 --> 01:03:36,950 REALLY QUICK. 1735 01:03:36,950 --> 01:03:39,285 WE TALKED ABOUT ASSUMPTIONS, HOW 1736 01:03:39,285 --> 01:03:44,691 DO I GET ON PAPER TO GO PRE-IND? 1737 01:03:44,691 --> 01:03:45,992 THERE ARE BASIC THINGS WE WANT 1738 01:03:45,992 --> 01:03:48,127 TO PUT ON PAPER AND THAT IS WHAT 1739 01:03:48,127 --> 01:03:51,331 THIS LIST IS HERE I A BASIC LIS 1740 01:03:51,331 --> 01:03:53,166 OF YOU WOULD WANT TO PUT 1741 01:03:53,166 --> 01:03:54,634 ON PAPER TO HAVE THAT FIRST 1742 01:03:54,634 --> 01:03:56,302 OUTLINE. 1743 01:03:56,302 --> 01:03:57,370 ON PAPER. 1744 01:03:57,370 --> 01:03:59,439 SO THE POINT P THIS SLIDE IS 1745 01:03:59,439 --> 01:04:02,242 THAT EARLIER THERE'S ALL THIS 1746 01:04:02,242 --> 01:04:03,276 BRAINSTORMING HAPPENING AND ALL 1747 01:04:03,276 --> 01:04:05,011 THESE DISCUSSIONS AND 1748 01:04:05,011 --> 01:04:06,646 ASSUMPTIONS AND YOU HAVE FIGURED 1749 01:04:06,646 --> 01:04:08,448 OUT THE TREATMENT JOURNEY OF THE 1750 01:04:08,448 --> 01:04:10,750 PATIENTS AND ALL OF THAT, THE 1751 01:04:10,750 --> 01:04:12,185 HARDEST PART TO ME SOMETIMES IS 1752 01:04:12,185 --> 01:04:14,387 NOW HOW DO WE ACTUALLY PUT THIS 1753 01:04:14,387 --> 01:04:17,156 ON PAPER IN A WAY THAT IT'S GOT 1754 01:04:17,156 --> 01:04:18,925 SCIENTIFIC AND CLINICAL MERIT? 1755 01:04:18,925 --> 01:04:20,760 THIS IS A REALLY GOOD OUTLINE 1756 01:04:20,760 --> 01:04:22,462 THAT HELPS PRIORITIZE THE TOPICS 1757 01:04:22,462 --> 01:04:24,697 YOU NEED TO FOCUS ON FIRST WHEN 1758 01:04:24,697 --> 01:04:27,000 YOU ARE TRYING TO DESIGN YOUR 1759 01:04:27,000 --> 01:04:27,934 CLINICAL TRIAL AND PUT IT ON 1760 01:04:27,934 --> 01:04:29,535 PAPER. 1761 01:04:29,535 --> 01:04:32,238 NEXT SLIDE. 1762 01:04:32,238 --> 01:04:35,141 SO CLINICAL TRIAL EXECUTION. 1763 01:04:35,141 --> 01:04:38,645 SO YOU CAN GO TO THE NEXT SLIDE. 1764 01:04:38,645 --> 01:04:40,446 IT IS JUST ONE SLIDE. 1765 01:04:40,446 --> 01:04:42,415 HOWEVER, WHILE THIS IS ONLY ONE 1766 01:04:42,415 --> 01:04:44,884 SLIDE, THERE ARE SO MANY MOVING 1767 01:04:44,884 --> 01:04:46,419 PIECES IN TRIAL PLANNING AND 1768 01:04:46,419 --> 01:04:47,086 EXECUTION. 1769 01:04:47,086 --> 01:04:48,388 MANY TIMES PEOPLE DON'T REALIZE 1770 01:04:48,388 --> 01:04:50,957 ALL OF THAT EARLY STRATEGY I WAS 1771 01:04:50,957 --> 01:04:53,593 TALKING ABOUT AND PLANNING 1772 01:04:53,593 --> 01:04:54,961 SOMETIMES, YOU KNOW, PEOPLE 1773 01:04:54,961 --> 01:04:56,696 OVERLOOK THE FACT THAT THIS 1774 01:04:56,696 --> 01:04:58,531 HEAVILY INFLUENCED THE CLINICAL 1775 01:04:58,531 --> 01:05:01,100 TRIAL DESIGN AND EXECUTION 1776 01:05:01,100 --> 01:05:01,567 STRATEGY. 1777 01:05:01,567 --> 01:05:03,069 SO IT ALWAYS IS IMPORTANT TO 1778 01:05:03,069 --> 01:05:05,305 HAVE A CLINICAL OPERATIONS 1779 01:05:05,305 --> 01:05:06,939 PERSON OR MEMBER AS PART OF 1780 01:05:06,939 --> 01:05:08,908 THOSE EARLIER EXERCISES BECAUSE 1781 01:05:08,908 --> 01:05:11,311 THEN THAT PERSON HAS TO FIGURE 1782 01:05:11,311 --> 01:05:14,814 OU TO SET UP THE TRIAL AND 1783 01:05:14,814 --> 01:05:15,748 IMPLEMENT IT. 1784 01:05:15,748 --> 01:05:17,417 AND SO WHEN USING THAT HOUSE 1785 01:05:17,417 --> 01:05:18,685 BUILDING SCENARIO, THIS IS WHERE 1786 01:05:18,685 --> 01:05:22,121 YOU PICK YOUR CABINETS, YOUR 1787 01:05:22,121 --> 01:05:23,723 FLOORS, THE FINE DETAILS LIKE 1788 01:05:23,723 --> 01:05:26,993 THE DOORKNOBS, WHAT COLOR DOORS 1789 01:05:26,993 --> 01:05:28,828 YOU WANT, WHAT STYLE CABINETS 1790 01:05:28,828 --> 01:05:29,862 AND COUNTER TOPS. 1791 01:05:29,862 --> 01:05:31,097 SOME OF THESE THINGS YOU CAN 1792 01:05:31,097 --> 01:05:35,401 CHANGE LATER, BUT IT WOULD I OUY 1793 01:05:35,401 --> 01:05:36,936 IMPORTANT YOU HAVE A GOOD IDEA 1794 01:05:36,936 --> 01:05:40,106 OF THAT PLANNING, THAT BLUEPRINT 1795 01:05:40,106 --> 01:05:42,275 AND YOU CAN DO THE FINISHING 1796 01:05:42,275 --> 01:05:42,508 TOUCHES. 1797 01:05:42,508 --> 01:05:44,777 THAT IS HOW I LOOK AT THIS 1798 01:05:44,777 --> 01:05:45,044 SCENARIO. 1799 01:05:45,044 --> 01:05:46,312 SO FOR EXAMPLE WHEN MAPPING OUT 1800 01:05:46,312 --> 01:05:48,748 THE REAL LIFE JOURNEY WE LEARN 1801 01:05:48,748 --> 01:05:51,084 WHAT TYPE OF PI YOU NEED TO GO 1802 01:05:51,084 --> 01:05:52,151 TO, WHAT SPECIALTY. 1803 01:05:52,151 --> 01:05:53,986 WHAT SITE SETTING YOU NEED TO 1804 01:05:53,986 --> 01:05:56,155 LOOK FOR SO YOU CAN KNOW WHAT 1805 01:05:56,155 --> 01:05:59,025 SITES TO IDENTIFY? 1806 01:05:59,025 --> 01:06:01,994 WHAT OPERATIONAL ELEMENTS ARE 1807 01:06:01,994 --> 01:06:02,495 NEEDED IN PLACE? 1808 01:06:02,495 --> 01:06:05,064 THE CRITICAL TRANSLATION PLAN 1809 01:06:05,064 --> 01:06:05,531 TELLS YOU WHAT 1810 01:06:05,531 --> 01:06:06,833 VENDORS YOU 1811 01:06:06,833 --> 01:06:07,900 NEED TO PICK. 1812 01:06:07,900 --> 01:06:09,702 SO THIS IS WHY IT IS IMPS 1813 01:06:09,702 --> 01:06:10,903 FO INDIVIDUALS THAT ARE 1814 01:06:10,903 --> 01:06:13,172 INVOLVED IN THE CLINICAL TRIAL 1815 01:06:13,172 --> 01:06:14,774 EXECUTION STRATEGY THAT THEY ARE 1816 01:06:14,774 --> 01:06:17,844 ALSO PARTICIPATING IN THE EARLY 1817 01:06:17,844 --> 01:06:18,144 DISCUSSIONS. 1818 01:06:18,144 --> 01:06:21,381 FOR CLINICAL TRIAL EXECUTION 1819 01:06:21,381 --> 01:06:22,982 STRATEGY THERE ARE ELEMENTS 1820 01:06:22,982 --> 01:06:25,551 PEOPLE DON'T THINK ABOUT THAT 1821 01:06:25,551 --> 01:06:28,955 MAY HEAVILY ENFORCE EXECUTION. 1822 01:06:28,955 --> 01:06:30,156 A COMPANY'S COMMERCIAL STRATEGY. 1823 01:06:30,156 --> 01:06:34,727 I ALWAYS TRYO , YOU 1824 01:06:34,727 --> 01:06:36,562 KNOW, WE'RE NOT ALWAYS WANTING A 1825 01:06:36,562 --> 01:06:38,331 BUSINESS TO TELL US HOW TO RUN 1826 01:06:38,331 --> 01:06:40,066 OUR TRIALS, HOWEVER THERE MAY BE 1827 01:06:40,066 --> 01:06:41,667 A COMMERCIAL STRATEGY THAT 1828 01:06:41,667 --> 01:06:43,770 GUIDES US INTO WHAT COUNTRIES WE 1829 01:06:43,770 --> 01:06:45,872 SHOULD GO INTO. 1830 01:06:45,872 --> 01:06:47,106 THEN AT THE SAME TIME AS A 1831 01:06:47,106 --> 01:06:48,374 CLINICAL OPERATION PERSON YOU 1832 01:06:48,374 --> 01:06:50,977 MAY BE THINKING WHERE ARE YOUR 1833 01:06:50,977 --> 01:06:52,245 PATIENTS HEAT MAP WISE? 1834 01:06:52,245 --> 01:06:53,379 THERE IS A DIFFICULT BALANCE 1835 01:06:53,379 --> 01:06:55,648 SOMETIMES TO FIGURE OUT HOW TO 1836 01:06:55,648 --> 01:06:57,817 BAKE IN ALL OF THE ELEMENTS 1837 01:06:57,817 --> 01:07:01,287 WHETHER IT IS COMMERCIAL, MAYBE 1838 01:07:01,287 --> 01:07:04,957 CMC THAT CAN'T GET KITS BY A 1839 01:07:04,957 --> 01:07:09,295 CERTAIN TIME OR I.V.BOLUS BUT 1840 01:07:09,295 --> 01:07:11,831 YOU PREFER A LONGER I.V. 1841 01:07:11,831 --> 01:07:13,800 THESE THINGS FEED INTO CLINICAL 1842 01:07:13,800 --> 01:07:14,167 TRIAL EXECUTION. 1843 01:07:14,167 --> 01:07:17,103 WHEN YOU ARE MAPPING OUT YOUR 1844 01:07:17,103 --> 01:07:19,105 TIMELINES THERE MAYBIN FLEXION 1845 01:07:19,105 --> 01:07:20,506 POINTS THAT ARE CRITICAL PATHS 1846 01:07:20,506 --> 01:07:22,508 FOR THE COMPANY OR CRITICAL PATH 1847 01:07:22,508 --> 01:07:24,911 TO BE ABLE TO INFORM WHETHER WE 1848 01:07:24,911 --> 01:07:27,413 DO A BID DOSE OR A COMBINATION 1849 01:07:27,413 --> 01:07:32,718 OR WHETHER IT IS SAFE FOR THE 1850 01:07:32,718 --> 01:07:32,952 PATIENT. 1851 01:07:32,952 --> 01:07:33,953 AYS COMES P FIRST, 1852 01:07:33,953 --> 01:07:37,089 WE NEED TO FACTOR IN THESE 1853 01:07:37,089 --> 01:07:38,458 ELEMENTS AND THE CLINICAL TRIAL 1854 01:07:38,458 --> 01:07:39,525 EXECUTION AND HOW WE ARE GOING 1855 01:07:39,525 --> 01:07:40,359 TO SET IT I. 1856 01:07:40,359 --> 01:07:41,828 THE LAST POINTS I'M GOING TO 1857 01:07:41,828 --> 01:07:43,729 MAKE IS WE HAVE GONE BACK AND 1858 01:07:43,729 --> 01:07:47,366 FORTH OF HOW WE SEE RECRUITMENT 1859 01:07:47,366 --> 01:07:48,468 STRATEGIES AND TACTICS. 1860 01:07:48,468 --> 01:07:50,670 I LIKED THE WAY THE PREVIOUS 1861 01:07:50,670 --> 01:07:52,405 SPEAKER TALKED ABOUT RECRUITMENT 1862 01:07:52,405 --> 01:07:54,407 STRATEGIES AND TACTICS. 1863 01:07:54,407 --> 01:07:56,175 I ALWAYS ENCOURAGE MY CLIENTS TO 1864 01:07:56,175 --> 01:07:57,844 GO BACK TO THE PATIENT AND THE 1865 01:07:57,844 --> 01:07:58,978 TREATMENT JOURNEY MAPPING AND 1866 01:07:58,978 --> 01:08:00,646 PUT THE PATIENT IN THE CENTER 1867 01:08:00,646 --> 01:08:02,381 AND FIGURE OUT THE BEST 1868 01:08:02,381 --> 01:08:04,684 RESOURCES AND ENGAGEMENTS THAT 1869 01:08:04,684 --> 01:08:07,687 WILL BUILD HEALTHY 1870 01:08:07,687 --> 01:08:09,222 SELF-AWARENESS ABOUT THE TRIAL. 1871 01:08:09,222 --> 01:08:10,389 ENGAGING PATIENT FOUNDATION AND 1872 01:08:10,389 --> 01:08:12,925 ADVOCACY GROUPS EARLY TO HELP A 1873 01:08:12,925 --> 01:08:14,093 DEVELOPER TO GET MORE CONNECTED 1874 01:08:14,093 --> 01:08:15,962 TO THE COMMUNITY, BUT MORE 1875 01:08:15,962 --> 01:08:16,829 IMPORTANTLY UNDERSTANDING THE 1876 01:08:16,829 --> 01:08:20,333 PATIENT BETTER AND SETTING UP AN 1877 01:08:20,333 --> 01:08:21,400 OPTIMAL PATIENT RECRUITMENT 1878 01:08:21,400 --> 01:08:24,370 RETENTION TOOLS THAT HELP THE 1879 01:08:24,370 --> 01:08:26,038 PATIENT NAVIGATE THROUGH THE 1880 01:08:26,038 --> 01:08:27,406 NUANCES AND CHALLENGES OF 1881 01:08:27,406 --> 01:08:28,474 PARTICIPATING IN A TRIAL. 1882 01:08:28,474 --> 01:08:30,643 I THINK WE DO ALL THESE THINGS, 1883 01:08:30,643 --> 01:08:33,913 WE WANT GOOD DATA, WE WANT THE 1884 01:08:33,913 --> 01:08:35,581 TRIAL TO BE, YOU KNOW, EASY FOR 1885 01:08:35,581 --> 01:08:37,350 THE SITE TO BE ABLE TO 1886 01:08:37,350 --> 01:08:39,552 IMPLEMENT, NOT MAKE A LOT OF 1887 01:08:39,552 --> 01:08:40,453 MISTAKES, ALL THESE THINGS. 1888 01:08:40,453 --> 01:08:42,922 AT THE END OF THE DAY, I TRY TO 1889 01:08:42,922 --> 01:08:46,759 ALWAYS PUT THE PATIENT FIRST AND 1890 01:08:46,759 --> 01:08:47,960 SAY, YOU KNOW, HOW DOES THIS, 1891 01:08:47,960 --> 01:08:49,996 YOU KNOW, HOW WOULD THE PATIENT 1892 01:08:49,996 --> 01:08:51,063 NAVIGATE THIS TRIAL? 1893 01:08:51,063 --> 01:08:53,232 WHAT IS GOING TO MAKE IT 1894 01:08:53,232 --> 01:08:53,533 IMPOSSIBLE? 1895 01:08:53,533 --> 01:08:55,234 YOU KNOW, WHEN WE ARE THINKING 1896 01:08:55,234 --> 01:08:58,371 ABOUT FRESH BIOPSIESIEIEERSUS 1897 01:08:58,371 --> 01:09:01,440 GETTING AN HISTORICAL REPORT. 1898 01:09:01,440 --> 01:09:03,242 THESE KINDS OF THINGS REALLY 1899 01:09:03,242 --> 01:09:05,511 MATTER AND BALANCING OUT YOUR 1900 01:09:05,511 --> 01:09:06,913 SCIENTIFIC MERIT AND THE 1901 01:09:06,913 --> 01:09:07,647 CLINICAL QUESTIONS YOU NEED TO 1902 01:09:07,647 --> 01:09:10,149 ANSWER IN YOUR TRIAL. 1903 01:09:10,149 --> 01:09:12,485 AND SOHESE THINGS ARE 1904 01:09:12,485 --> 01:09:14,186 EXTREMELY IMPORTANT WHEN YOU ARE 1905 01:09:14,186 --> 01:09:15,454 THINKING ABOUT OPERATIONAL 1906 01:09:15,454 --> 01:09:16,756 PLANNING AND EXECUTION. 1907 01:09:16,756 --> 01:09:23,462 SO THAT'S THE END OF MYPRESENTA. 1908 01:09:23,462 --> 01:09:24,730 >> THANKS SO MUCH, DORIS. 1909 01:09:24,730 --> 01:09:27,567 WHAT A GREAT SUMMARY OF A VERY, 1910 01:09:27,567 --> 01:09:28,568 VERY COMPLICATED PROCESS. 1911 01:09:28,568 --> 01:09:30,269 NICE WORK TO DO THAT IN 20 1912 01:09:30,269 --> 01:09:32,972 MINUTES. 1913 01:09:32,972 --> 01:09:36,542 WE'LL MOVE ON TO D DETERDING 1914 01:09:36,542 --> 01:09:40,046 TO TELL US HOW THIS PROCESS WENT 1915 01:09:40,046 --> 01:09:42,281 FOR HER IN HER TRIAL. 1916 01:09:42,281 --> 01:09:42,915 >> HI, EVERYBODY. 1917 01:09:42,915 --> 01:09:46,452 I'M EXCITED TO TALK WITH YOU W 1918 01:09:46,452 --> 01:09:48,054 THROUGH THINKING ABOUT CHILDREN 1919 01:09:48,054 --> 01:09:50,089 WITH RARE LUNG DISEASE. 1920 01:09:50,089 --> 01:09:52,692 AND AS PART OF THIS, I WILL 1921 01:09:52,692 --> 01:09:55,328 MENTION WAYS THAT WE'VE HAD TO 1922 01:09:55,328 --> 01:09:56,729 ADAPT, BARRIERS AND SOLUTIONS 1923 01:09:56,729 --> 01:09:59,098 THAT WE ARRIVED AT THAT OTHER 1924 01:09:59,098 --> 01:09:59,699 PEOPLE CAN THINK ABOUT. 1925 01:09:59,699 --> 01:10:04,670 NEXT. 1926 01:10:04,670 --> 01:10:07,206 I DO HAVE SOME CONFLICT OF 1927 01:10:07,206 --> 01:10:12,545 INTEREST RELEVANT FINANCIAL 1928 01:10:12,545 --> 01:10:18,951 RELATIONSHIPS AROUND BEOHRINGER 1929 01:10:18,951 --> 01:10:19,418 INND THE FRE 1930 01:10:19,418 --> 01:10:20,353 FOUNDATION AND THE REST YOU CAN 1931 01:10:20,353 --> 01:10:20,519 SEE. 1932 01:10:20,519 --> 01:10:22,888 NEXT. 1933 01:10:22,888 --> 01:10:25,958 SO I JUST WANTED TO START BY 1934 01:10:25,958 --> 01:10:27,960 PROVIDING THE CLINICIAN'S 1935 01:10:27,960 --> 01:10:29,662 PERSPECTIVE OF TREATMENT, 1936 01:10:29,662 --> 01:10:31,764 ESPECIALLY IN KIDS WITH RARE 1937 01:10:31,764 --> 01:10:33,399 LUNG DISEASE. 1938 01:10:33,399 --> 01:10:37,303 AND THIS IS WHAT WE DO. 1939 01:10:37,303 --> 01:10:39,271 SOME OF THE CLINICIANS ON WILL 1940 01:10:39,271 --> 01:10:40,172 UNDERSTAND THIS. 1941 01:10:40,172 --> 01:10:41,574 WE USE OFF LABEL DRUGS. 1942 01:10:41,574 --> 01:10:43,709 THIS IS OUR STANDARD WAY WE 1943 01:10:43,709 --> 01:10:44,610 APPROACH THINGS. 1944 01:10:44,610 --> 01:10:46,846 MOST DRUGS AREN'T STUDIED IN 1945 01:10:46,846 --> 01:10:47,113 CHILDREN. 1946 01:10:47,113 --> 01:10:48,648 THE BENEFIT IS THEYRE 1947 01:10:48,648 --> 01:10:50,082 AVAILABLE, WE HAVE INSIGHTS FROM 1948 01:10:50,082 --> 01:10:51,384 MMON THING.MENTUL MOST 1949 01:10:51,384 --> 01:10:53,653 THERE ARE A LOT OF RISKS. 1950 01:10:53,653 --> 01:10:56,489 WE DON'T KNOW INFORMATION ABOUT 1951 01:10:56,489 --> 01:10:58,891 DOSING, PROFILE ON KIDS, 1952 01:10:58,891 --> 01:11:00,059 SOMETIMES THEY ARE NOT COVERED 1953 01:11:00,059 --> 01:11:03,829 BY INSURANCE AND SOMETIMES 1954 01:11:03,829 --> 01:11:04,096 UNPROVEN. 1955 01:11:04,096 --> 01:11:06,532 WE GET TO CLINICAL TRIALS WITH 1956 01:11:06,532 --> 01:11:08,367 DRUGS AVAILABLE IN THE ADULT 1957 01:11:08,367 --> 01:11:11,637 MARKET THIS HAPPENS, THERE IS 1958 01:11:11,637 --> 01:11:16,175 NOW A MOVEMENT BY FDA AND EMA T 1959 01:11:16,175 --> 01:11:17,910 CONSIDERATION OF DRUGS AND WE'LL 1960 01:11:17,910 --> 01:11:19,245 TALK ABOUT SOME OF THAT. 1961 01:11:19,245 --> 01:11:20,846 THAT LED US TO THE TENTATIVE 1962 01:11:20,846 --> 01:11:24,083 TRIAL FOR KIDS WITH PULMONARY 1963 01:11:24,083 --> 01:11:24,517 FIBROSIS. 1964 01:11:24,517 --> 01:11:26,185 THESE DRUGS ARE USUALLY TESTED 1965 01:11:26,185 --> 01:11:27,219 IN ADULTS FIRST. 1966 01:11:27,219 --> 01:11:29,388 THERE IS ADULT DATA AND THE 1967 01:11:29,388 --> 01:11:30,523 REGULATORY REQUIREMENTS INCLUDE 1968 01:11:30,523 --> 01:11:32,291 THERE SHOULD BE A PEDIATRIC 1969 01:11:32,291 --> 01:11:33,292 INVESTIGATION PLAN. 1970 01:11:33,292 --> 01:11:36,028 THIS IS OPENED UP OPPORTUNITIES 1971 01:11:36,028 --> 01:11:39,098 FOR KIDS TO HAVE DRUGS STUDIED 1972 01:11:39,098 --> 01:11:40,399 AND BRING DRUGS FORWARD, 1973 01:11:40,399 --> 01:11:43,002 HOWEVER, IT IS A VERY EXPENSIVE 1974 01:11:43,002 --> 01:11:43,269 ENDEAVOR. 1975 01:11:43,269 --> 01:11:44,970 WE HAVE FEW PATIENTS THAT HAVE 1976 01:11:44,970 --> 01:11:47,139 TO GO THROUGH REGULATORY 1977 01:11:47,139 --> 01:11:49,241 APPROVAL AND IT TAKES TIME. 1978 01:11:49,241 --> 01:11:50,810 BUT I'M GOING TO BE TALKING 1979 01:11:50,810 --> 01:11:51,811 ABOUT THAT TODAY. 1980 01:11:51,811 --> 01:11:54,146 THEN THERE'S NEW DISCOVERY. 1981 01:11:54,146 --> 01:11:56,682 THERE HAS BEEN SOME EXCITING 1982 01:11:56,682 --> 01:11:56,916 SCIENCE. 1983 01:11:56,916 --> 01:11:58,751 MORE MECHANICISTICS, GET TO 1984 01:11:58,751 --> 01:11:59,485 CURING DISEASE. 1985 01:11:59,485 --> 01:12:04,023 IT IS SO EXCITING, IT CAN BE 1986 01:12:04,023 --> 01:12:05,257 RISKY, EXPENSIVE. 1987 01:12:05,257 --> 01:12:07,493 I HOPE WE HAVE NEW MODELS, BUT 1988 01:12:07,493 --> 01:12:08,861 IT CAN TAKE A LONG TIME. 1989 01:12:08,861 --> 01:12:10,863 THIS IS CURRENTLY AS A CLINICIAN 1990 01:12:10,863 --> 01:12:13,332 WHAT WE LOOK AT AND AS AN 1991 01:12:13,332 --> 01:12:15,034 ADVOCATE FORCAG CAREF KIDS 1992 01:12:15,034 --> 01:12:17,103 THAT WE DO.ISEASE THAT I SEE I 1993 01:12:17,103 --> 01:12:17,303 NEXT. 1994 01:12:17,303 --> 01:12:19,872 THAT SETS THE STAGE FOR HOW WE 1995 01:12:19,872 --> 01:12:22,975 ARRIVE AT THE FIRST CLINICAL 1996 01:12:22,975 --> 01:12:25,611 TRIAL FOR KIDS WITH REALLY 1997 01:12:25,611 --> 01:12:27,580 PULMONARY FIBROSIS. 1998 01:12:27,580 --> 01:12:29,949 THAT IS WHAT I WILL BE TALKING 1999 01:12:29,949 --> 01:12:30,483 ABOUT TODAY. 2000 01:12:30,483 --> 01:12:34,653 WE WERE APPROACHED ABOUT 2001 01:12:34,653 --> 01:12:36,555 CONDUCTING A TRIAL FOR CHILDREN 2002 01:12:36,555 --> 01:12:41,727 FOR THEIR PEDIATRIC 2003 01:12:41,727 --> 01:12:43,162 INVESTIGATIONAL PLAN FOR 2004 01:12:43,162 --> 01:12:44,096 ATTENTIVE. 2005 01:12:44,096 --> 01:12:45,097 OUR SCIENTIFIC COMMUNITY WAS 2006 01:12:45,097 --> 01:12:46,532 APPROACHED ABOUT RUNNING A 2007 01:12:46,532 --> 01:12:46,732 TRIAL. 2008 01:12:46,732 --> 01:12:48,200 IT WAS QUITE EYE OPENING BECAUSE 2009 01:12:48,200 --> 01:12:49,735 WE DON'T REALLY HAVE RESOURCES. 2010 01:12:49,735 --> 01:12:52,471 I WAS THINKING ABOUT THE LAST 2011 01:12:52,471 --> 01:12:54,173 SPEAKER, THINKING ABOUT YOUR 2012 01:12:54,173 --> 01:12:54,440 RESOURCES. 2013 01:12:54,440 --> 01:12:56,242 WE DON'T HAVE RESOURCES, REALLY, 2014 01:12:56,242 --> 01:12:57,076 LIMITED RESOURCES. 2015 01:12:57,076 --> 01:12:58,844 SO THIS WAS A GREAT OPPORTUNITY 2016 01:12:58,844 --> 01:13:00,446 TO THINK ABOUT MOVING THE FIELD 2017 01:13:00,446 --> 01:13:01,413 FORWARD FOR KIDS. 2018 01:13:01,413 --> 01:13:03,549 THERE WAS A LOT WE DIDN'T KNOW 2019 01:13:03,549 --> 01:13:04,850 WHEN WE SAT DOWN. 2020 01:13:04,850 --> 01:13:06,685 TALKING ABOUT BARRIERS AND 2021 01:13:06,685 --> 01:13:08,320 TALKING ABOUT WHAT CAN HAPPEN 2022 01:13:08,320 --> 01:13:08,554 THOUGH. 2023 01:13:08,554 --> 01:13:10,189 WE HAD A WHOLE LIST OF THINGS WE 2024 01:13:10,189 --> 01:13:11,824 HAD TO THINK ABOUT TO DEFINE 2025 01:13:11,824 --> 01:13:12,758 THIS STUDY. 2026 01:13:12,758 --> 01:13:15,394 WE HAD TO DEFINE WHAT FIBROSIS 2027 01:13:15,394 --> 01:13:16,562 WAS IN CHILDREN. 2028 01:13:16,562 --> 01:13:19,565 WHAT EXACERBATIONS WERE, WHAT 2029 01:13:19,565 --> 01:13:22,401 PEDIATRIC CHILD PROGRESSIVE 2030 01:13:22,401 --> 01:13:23,002 FIBROSIS DISEASES WOULD BE 2031 01:13:23,002 --> 01:13:24,370 NATURAL HISTORY. 2032 01:13:24,370 --> 01:13:27,373 WE DON'T HAVE -- WE ARE EVOLVING 2033 01:13:27,373 --> 01:13:28,374 REGISTRIES, BUT NATURAL HISTORY 2034 01:13:28,374 --> 01:13:29,942 IS SO IMPORTANT. 2035 01:13:29,942 --> 01:13:31,677 AND THAT WAS SOMEWHAT MISSING. 2036 01:13:31,677 --> 01:13:33,445 YOU CAN SEE THE WHOLE LIST. 2037 01:13:33,445 --> 01:13:35,781 BASICALLY HAD TO START FROM 2038 01:13:35,781 --> 01:13:38,317 SCRATCH TO GET DOWN TO DIVINE 2039 01:13:38,317 --> 01:13:39,819 THIS STUDY AND THIS TRIAL. 2040 01:13:39,819 --> 01:13:42,388 SO THIS WAS A LEAP OF FAITH FROM 2041 01:13:42,388 --> 01:13:44,156 TO DESIGN A 2042 01:13:44,156 --> 01:13:46,725 TRIAL TO GET T WHERE WE ENDED 2043 01:13:46,725 --> 01:13:47,960 UP, WHICH I'M VERY PROUD OF. 2044 01:13:47,960 --> 01:13:49,962 ONE OF THE THINGS I WILL TALK 2045 01:13:49,962 --> 01:13:52,031 ABOUT TODAY, NEXT, WE NEED TO 2046 01:13:52,031 --> 01:13:58,437 THINK ABOUT DIFFERENCES BETWEEN 2047 01:13:58,437 --> 01:14:02,241 ADULT AND CHILD PULMONARY 2048 01:14:02,241 --> 01:14:03,108 FIBROSIS. 2049 01:14:03,108 --> 01:14:03,309 NEXT. 2050 01:14:03,309 --> 01:14:06,078 THE PREVIOUS SLIDE GAVE A LOT OF 2051 01:14:06,078 --> 01:14:07,646 BARRIERS IF YOU DON'T KNOW THOSE 2052 01:14:07,646 --> 01:14:07,880 THINGS. 2053 01:14:07,880 --> 01:14:10,616 WE HAD TO WORK THROUGH ALL 2054 01:14:10,616 --> 01:14:10,816 THOSE. 2055 01:14:10,816 --> 01:14:13,452 THE FIRST THING IS THERE A NEED? 2056 01:14:13,452 --> 01:14:16,155 ARE THERE CONDITIONS WITH 2057 01:14:16,155 --> 01:14:17,523 FIBROSIS IN PEDIATRIC 2058 01:14:17,523 --> 01:14:18,424 INTERSTITIAL LUNG DISEASE? 2059 01:14:18,424 --> 01:14:22,027 THE ANSWER IS THERE ARE. 2060 01:14:22,027 --> 01:14:25,731 YOU HEART ABOUT SURFACTANT 2061 01:14:25,731 --> 01:14:30,536 PROTEN DEFICIENCY AND THERE I A 2062 01:14:30,536 --> 01:14:33,405 LIST OF KIDS WITH FIBROSIS THAT 2063 01:14:33,405 --> 01:14:36,275 INCLUDE HIGH MORBIDITY, HIGH 2064 01:14:36,275 --> 01:14:37,776 MORTALITY CONDITIONS. 2065 01:14:37,776 --> 01:14:40,679 PULMONARY FIBROSIS IS ALL OF 2066 01:14:40,679 --> 01:14:42,481 THAT AND WE HAVE VERY FEW 2067 01:14:42,481 --> 01:14:44,650 TREATMENT OPTIONS. 2068 01:14:44,650 --> 01:14:44,850 NEXT. 2069 01:14:44,850 --> 01:14:47,720 SO SPEAKING OF WHAT WE DO AS 2070 01:14:47,720 --> 01:14:49,955 CLINICIANS, AS I TOLD YOU, WELL, 2071 01:14:49,955 --> 01:14:52,858 WE USE OFF-LABEL TREATMENT AND 2072 01:14:52,858 --> 01:14:54,593 ANECDOTAL TREATMENT APPROACHES. 2073 01:14:54,593 --> 01:15:00,599 THIS IS THE CURRENT APPROACH. 2074 01:15:00,599 --> 01:15:04,270 THAT WE APPROACH KIDS WITH 2075 01:15:04,270 --> 01:15:05,971 INTERSTITIAL LUNG DISEASE OR 2076 01:15:05,971 --> 01:15:09,541 PULMONARY FIBROFIBROSIS. 2077 01:15:09,541 --> 01:15:10,476 THE WHOLE COMMUNITY DOES THE 2078 01:15:10,476 --> 01:15:13,112 SAME THING BUT NONE OF THIS ISF 2079 01:15:13,112 --> 01:15:13,545 STUDIED. 2080 01:15:13,545 --> 01:15:15,714 WE THEN THINK ABOUT MED 2081 01:15:15,714 --> 01:15:17,549 DISEASES AND WE HAVE OTHER SET 2082 01:15:17,549 --> 01:15:22,154 OF MEDICATIONS THAT HAVE BEEN 2083 01:15:22,154 --> 01:15:31,297 EVOLVING IN THE RHEUMATOLOGIC 2084 01:15:31,297 --> 01:15:32,498 SPACE. 2085 01:15:32,498 --> 01:15:36,468 NONE OF THESE HAVE BEEN 2086 01:15:36,468 --> 01:15:38,771 EXCLUSIVELY STUDIED IN S CHILDR. 2087 01:15:38,771 --> 01:15:39,238 THIS CAU 2088 01:15:39,238 --> 01:15:39,838 GREAT PAUSE. 2089 01:15:39,838 --> 01:15:43,842 WE LEARN A LOT FROM OUR ADULT 2090 01:15:43,842 --> 01:15:44,143 COLLEAGUES. 2091 01:15:44,143 --> 01:15:46,312 OUR ADULT COLLEAGUES HAVE MORE 2092 01:15:46,312 --> 01:15:46,578 PATIENTS. 2093 01:15:46,578 --> 01:15:47,880 WE PAY ATTENTION IN WHAT HAPPENS 2094 01:15:47,880 --> 01:15:49,648 TO THEM TO BRING THE BEST TO 2095 01:15:49,648 --> 01:15:49,915 CHILDREN. 2096 01:15:49,915 --> 01:15:54,954 THIS STUDY HIGHLIGHTS WHY WE 2097 01:15:54,954 --> 01:15:58,123 NEED PLANS FOR KIDS TO HAVE DRIG 2098 01:15:58,123 --> 01:15:58,357 STUDIES. 2099 01:15:58,357 --> 01:16:00,159 THIS STUDY, THE PANTHER TRIAL 2100 01:16:00,159 --> 01:16:02,594 WAS CONDUCTED IN ADULTS WITH 2101 01:16:02,594 --> 01:16:03,529 PULMONARY IS. 2102 01:16:03,529 --> 01:16:05,864 WHAT THEY LOOKED AT IS NOW THIS 2103 01:16:05,864 --> 01:16:08,100 IS IN 2012, SO IT WAS A WHILE 2104 01:16:08,100 --> 01:16:08,500 AGO. 2105 01:16:08,500 --> 01:16:11,270 THEY LOOKED AT THEIR STANDARD OF 2106 01:16:11,270 --> 01:16:19,945 CARE AT THE TIME WAS PREDNISONE 2107 01:16:19,945 --> 01:16:22,848 AND -- THE RESULTS WERE VERY EYE 2108 01:16:22,848 --> 01:16:23,115 OPENING. 2109 01:16:23,115 --> 01:16:24,717 IN ALMOST EVERY OUTCOME END 2110 01:16:24,717 --> 01:16:26,552 POINT FROM DEATH TO 2111 01:16:26,552 --> 01:16:30,823 HOSPITALIZATION TO 2112 01:16:30,823 --> 01:16:33,192 EXACERBAHE TREATMENT ARM 2113 01:16:33,192 --> 01:16:35,060 WAS THE HIGHEST RISK FOR DEATH, 2114 01:16:35,060 --> 01:16:36,328 FOR ALL OF THAT. 2115 01:16:36,328 --> 01:16:39,465 AND I THINK THAT HIGHLIGHTS THAT 2116 01:16:39,465 --> 01:16:41,233 WE HAVE TO DO MORE, WE HAVE TO 2117 01:16:41,233 --> 01:16:43,302 DO THESE STUDIES FOR KIDS. 2118 01:16:43,302 --> 01:16:45,337 WE HAVE TO ADVOCATE THAT THESE 2119 01:16:45,337 --> 01:16:45,871 THINGS HAPPEN. 2120 01:16:45,871 --> 01:16:47,439 WE USE SOME OF THESE MEDICATIONS 2121 01:16:47,439 --> 01:16:47,673 IN KIDS. 2122 01:16:47,673 --> 01:16:51,810 NEXT. 2123 01:16:51,810 --> 01:16:53,412 SO IT CAUSES US TO LOOK TO WHAT 2124 01:16:53,412 --> 01:16:56,949 IS HAPPENING IN THE ADULT REALM. 2125 01:16:56,949 --> 01:16:58,884 KIDS WITH PULMONARY FIBROSIS, 2126 01:16:58,884 --> 01:17:00,786 MEDICATIONS HAVE BEEN APPROVED 2127 01:17:00,786 --> 01:17:04,223 SINCE 2014. 2128 01:17:04,223 --> 01:17:05,991 BIG PHASE 3 TRIALS HAVE BEEN 2129 01:17:05,991 --> 01:17:06,325 DONE. 2130 01:17:06,325 --> 01:17:08,293 SO THE QUESTION WE ASK IS HOW DO 2131 01:17:08,293 --> 01:17:10,195 WE GET ACCESS TO THESE TRIALS? 2132 01:17:10,195 --> 01:17:12,431 HOW DO WE GET ACCESS TO THESE 2133 01:17:12,431 --> 01:17:14,233 DRUGS FROM A TRIAL? 2134 01:17:14,233 --> 01:17:15,367 OUR COMMUNITY HAS BEEN ASKING 2135 01:17:15,367 --> 01:17:15,734 THESE QUESTIONS. 2136 01:17:15,734 --> 01:17:23,208 NEXT. 2137 01:17:23,208 --> 01:17:29,915 SO THE TRIALS HAVE INCLUDEDLU 2138 01:17:29,915 --> 01:17:32,518 DIOPATHIC PULMONARY FIBROSIS 2139 01:17:32,518 --> 01:17:33,452 STUDIES. 2140 01:17:33,452 --> 01:17:36,688 SYSTEMIC SCLEROSIS SOERTED ILD 2141 01:17:36,688 --> 01:17:40,526 AND PROGRESSIVE FIBROSING ILD. 2142 01:17:40,526 --> 01:17:44,196 THE COMMUNITY SAID, HEY, THESE 2143 01:17:44,196 --> 01:17:47,666 DISEASE STUDIES FOR PULMONARY 2144 01:17:47,666 --> 01:17:48,767 FIBROSIS, WE ARE NEVER GOING TO 2145 01:17:48,767 --> 01:17:50,803 GET TO PATIENTS WHO NEED HELP. 2146 01:17:50,803 --> 01:17:54,006 WE H PATIENTS THAT THESE 2147 01:17:54,006 --> 01:17:56,175 MECHC DRUGSTIFERE IN 2148 01:17:56,175 --> 01:18:00,245 THE FIBROTIC PATHWAY. 2149 01:18:00,245 --> 01:18:03,048 THEY DEVELOPED A STUDY WHERE 2150 01:18:03,048 --> 01:18:05,818 THEY TOOK ADULT CONDITIONS WITH 2151 01:18:05,818 --> 01:18:08,987 PROGRESSIVE FIBROTIC LUNG 2152 01:18:08,987 --> 01:18:10,989 DISEASE AND BASKETED THOSE 2153 01:18:10,989 --> 01:18:13,058 DISEASES INTO A COHORT TO STUDY. 2154 01:18:13,058 --> 01:18:14,493 THAT BECAME VERY IMPORTANT. 2155 01:18:14,493 --> 01:18:17,362 ALL OF THESEPREWORK WAS 2156 01:18:17,362 --> 01:18:18,730 CRITICALLY IMPORTA GETTING 2157 01:18:18,730 --> 01:18:20,365 TO A PEDIATRIC STUDY. 2158 01:18:20,365 --> 01:18:22,334 CO APPROACHED US, WE 2159 01:18:22,334 --> 01:18:23,936 APPROACHED THEM AND WERE ABLE TO 2160 01:18:23,936 --> 01:18:25,838 CONCEIVE OF THIS STUDY AND I'LL 2161 01:18:25,838 --> 01:18:26,672 EXPLAIN WHY. 2162 01:18:26,672 --> 01:18:26,939 NEXT. 2163 01:18:26,939 --> 01:18:30,342 SO THE GOOD NEWS IS IN ALL THE 2164 01:18:30,342 --> 01:18:32,845 STUDIES WHERE THIS DRUG WAS USED 2165 01:18:32,845 --> 01:18:34,813 IN DIFFERENT CONDITIONS, WHETHER 2166 01:18:34,813 --> 01:18:40,919 IT BE IPF, SYSTEMIC SCLEROSIS OR 2167 01:18:40,919 --> 01:18:43,689 THE INBUILD STUDY YOU SEE HERE, 2168 01:18:43,689 --> 01:18:47,893 THE DRUG SLOWED THE CHANGE FROM 2169 01:18:47,893 --> 01:18:48,160 BASELINE. 2170 01:18:48,160 --> 01:18:51,196 THE PRIMARY END POINT FOR MOST 2171 01:18:51,196 --> 01:18:52,831 REGULATORY APPROVAL PATHWAYS FOR 2172 01:18:52,831 --> 01:18:54,566 DRUGS. 2173 01:18:54,566 --> 01:19:00,572 IN THIS STUDY THE NINTENATIVE 2174 01:19:00,572 --> 01:19:03,408 GROUP, IPF OR ALL COMMERCE 2175 01:19:03,408 --> 01:19:06,712 SLOWED LUNG DECLINE WITH 2176 01:19:06,712 --> 01:19:07,546 FIBROSING ILDs. 2177 01:19:07,546 --> 01:19:09,681 THAT OCCURRED IN ALMOST ALL OF 2178 01:19:09,681 --> 01:19:11,583 THE PHASE 3 STUDIES. 2179 01:19:11,583 --> 01:19:15,053 NEXT. 2180 01:19:15,053 --> 01:19:17,356 SO WE SAID THIS INDICATIONS FOR 2181 01:19:17,356 --> 01:19:19,458 US TO STUDY THIS IN CHILDREN IS 2182 01:19:19,458 --> 01:19:24,429 WE BELIEVE THAT THE 2183 01:19:24,429 --> 01:19:25,998 PATHOPHYSIOLOGY O FIBROSIS IN 2184 01:19:25,998 --> 01:19:27,533 ADULTS AND CHILDREN ARE SIMILAR 2185 01:19:27,533 --> 01:19:29,268 ENOUGH WE SHOULD PROCEED. 2186 01:19:29,268 --> 01:19:30,969 YES, THERE IS A DEVELOPMENTAL 2187 01:19:30,969 --> 01:19:31,837 BACKGROUND FOR CHILDREN. 2188 01:19:31,837 --> 01:19:34,940 THE FIBROTIC MECHANISMS AND 2189 01:19:34,940 --> 01:19:36,575 PATHWAYS WOULD BE SIMILAR ENOUGH 2190 01:19:36,575 --> 01:19:39,011 FOR THIS MEDICATION TO BE 2191 01:19:39,011 --> 01:19:39,611 EFFECTIVE. 2192 01:19:39,611 --> 01:19:42,147 MODES OF ACTION THAT INHIBIT 2193 01:19:42,147 --> 01:19:43,282 PROGRESSIVE FIBROSIS WOULD BE 2194 01:19:43,282 --> 01:19:46,852 THE SAME. 2195 01:19:46,852 --> 01:19:51,423 ESTABLISHING CLINICAL BENEFIT OF 2196 01:19:51,423 --> 01:19:53,025 NINTEDANIB IN ADULTS COULD 2197 01:19:53,025 --> 01:19:54,960 HAPPEN SO WE COULD TAKE OUR 2198 01:19:54,960 --> 01:19:59,064 SMALL POPULATIONS AND COMBINE 2199 01:19:59,064 --> 01:20:02,601 THEN INTO ONE FIBROSING 2200 01:20:02,601 --> 01:20:03,569 PEDIATRIC POPULATION. 2201 01:20:03,569 --> 01:20:04,303 THESE WERE OUR CONSIDERATIONS. 2202 01:20:04,303 --> 01:20:05,704 NEXT. 2203 01:20:05,704 --> 01:20:08,073 AND SO THAT'S WHAT WE DID. 2204 01:20:08,073 --> 01:20:09,575 WE CAN DO THAT, TOO. 2205 01:20:09,575 --> 01:20:11,310 WE CAN USE THE BASKET APPROACH 2206 01:20:11,310 --> 01:20:12,244 IN KIDS. 2207 01:20:12,244 --> 01:20:14,780 I JUST SHOWED YOU THE LIST OF 2208 01:20:14,780 --> 01:20:16,815 PATIENTS THAT HAD PULMONARY 2209 01:20:16,815 --> 01:20:17,149 FIBROSIS. 2210 01:20:17,149 --> 01:20:19,952 WE CAN CREATE THAT SAME BASKET 2211 01:20:19,952 --> 01:20:21,620 APPROACH TO C T OURPOPULATION T. 2212 01:20:21,620 --> 01:20:30,963 NEXT. 2213 01:20:30,963 --> 01:20:31,163 NEXT. 2214 01:20:31,163 --> 01:20:31,363 UH-OH. 2215 01:20:31,363 --> 01:20:41,406 NE 2216 01:20:42,307 --> 01:20:46,345 COULD WE ADVANCE THE SLIDES, 2217 01:20:46,345 --> 01:20:46,578 PLEASE. 2218 01:20:46,578 --> 01:20:50,215 >> ANTONE, ARE YOU WITH US? 2219 01:20:50,215 --> 01:20:53,185 >> YES, ONE SECOND, MY COMPUTER 2220 01:20:53,185 --> 01:20:53,385 FROZE. 2221 01:20:53,385 --> 01:20:55,087 >> I'LL FILL THE GAP. 2222 01:20:55,087 --> 01:20:55,554 OKAY. 2223 01:20:55,554 --> 01:20:55,854 THANK YOU. 2224 01:20:55,854 --> 01:20:58,490 SO THIS LED TO A TRIAL THAT HAS 2225 01:20:58,490 --> 01:21:00,659 BEEN SUCCESSFULLY COMPLETED 2226 01:21:00,659 --> 01:21:07,165 WHERE WE LOOKED AT NINTEDANIB IN 2227 01:21:07,165 --> 01:21:10,002 CHILDREN AND ADULTS WITH 2228 01:21:10,002 --> 01:21:11,169 FIBROSING INTERSTITIAL LUNG 2229 01:21:11,169 --> 01:21:11,436 DISEASES. 2230 01:21:11,436 --> 01:21:12,904 I DON'T HAVE TIME TO PRESENT ALL 2231 01:21:12,904 --> 01:21:14,273 THE STUDY. 2232 01:21:14,273 --> 01:21:15,807 I'M GOING TO PRESENT HIGHLIGHTS 2233 01:21:15,807 --> 01:21:17,376 THAT ARE IMPORTANT TO THINK 2234 01:21:17,376 --> 01:21:18,710 ABOUT AS WE MOVE FORWARD WITH 2235 01:21:18,710 --> 01:21:19,978 KIDS WITH DIFFERENT TYPES OF 2236 01:21:19,978 --> 01:21:21,647 PROBLEMS AND PERHAPS THIS STUDY 2237 01:21:21,647 --> 01:21:23,448 CAN BE HELPFUL IN THINKING ABOUT 2238 01:21:23,448 --> 01:21:24,149 DESIGNINS FIN 2239 01:21:24,149 --> 01:21:33,525 SO OUR KEY INCLUSION CRITERIA 2240 01:21:33,525 --> 01:21:37,462 FOR THE INPED ILD TRIAL, 6-17 2241 01:21:37,462 --> 01:21:38,430 YEARS OF AGE. 2242 01:21:38,430 --> 01:21:46,638 EVIDENCING OF FIBROSING ILD ON 2243 01:21:46,638 --> 01:21:50,442 HRCT, FVC GREATER THAN 25%. 2244 01:21:50,442 --> 01:21:53,679 CLINICAL SCORING OF DISEASE. 2245 01:21:53,679 --> 01:21:54,813 PEDIATRICS WERE DIFFERENT FROM 2246 01:21:54,813 --> 01:21:55,414 ADULTS. 2247 01:21:55,414 --> 01:21:58,483 WE HAD TO DESIGN HOW KIDS COULD 2248 01:21:58,483 --> 01:21:59,551 MTS.DIFFERENT FROM 2249 01:21:59,551 --> 01:22:01,553 WE USED A PLAN SCORING SYSTEM 2250 01:22:01,553 --> 01:22:03,422 WHICH LOOKS AT HOW OXYGEN IS 2251 01:22:03,422 --> 01:22:04,122 USED IN CHILDREN. 2252 01:22:04,122 --> 01:22:05,991 THAT WAS PART OF OUR DESIGN. 2253 01:22:05,991 --> 01:22:11,063 THAT WAS A NEW DESIGN AND THE 2254 01:22:11,063 --> 01:22:15,434 CLASSIC MEASUREMENTS BY CAPACITY 2255 01:22:15,434 --> 01:22:18,370 THAT ARE STANDARD IN CLINICAL 2256 01:22:18,370 --> 01:22:19,705 MEASURES OF CLASSIC FIBROSIS. 2257 01:22:19,705 --> 01:22:22,574 SO THESE KIDS TO BE SI 2258 01:22:22,574 --> 01:22:22,908 NEXT. 2259 01:22:22,908 --> 01:22:25,077 ONE OF THE KEY BARRIERS WE GOT 2260 01:22:25,077 --> 01:22:27,379 TO ALMOST RIGHT AWAY IS THAT FOR 2261 01:22:27,379 --> 01:22:31,717 MANY OF THESE KIDS WITH 2262 01:22:31,717 --> 01:22:35,387 FIBROSIS, THE EASIEST WAY TO 2263 01:22:35,387 --> 01:22:37,756 DIAGNOSE FIBROSIS IS A LUNG 2264 01:22:37,756 --> 01:22:38,690 BIOPSY. 2265 01:22:38,690 --> 01:22:43,628 BUT AS MORE AND MORE OF THESE 2266 01:22:43,628 --> 01:22:45,697 FIBROTTIC CONDITIONS WERE 2267 01:22:45,697 --> 01:22:47,599 DIAGNOSED, THESE KIDS WERE NOT 2268 01:22:47,599 --> 01:22:48,834 GETTING LUNGES. 2269 01:22:48,834 --> 01:22:51,203 WEAD TO THINK OF OTHER 2270 01:22:51,203 --> 01:22:52,204 CRITERIA THAN THE GOLD STANDARD 2271 01:22:52,204 --> 01:22:53,405 OF LUNG BIOPSY. 2272 01:22:53,405 --> 01:22:56,541 THERE WAS NO C T CRITERIA FOR 2273 01:22:56,541 --> 01:22:58,343 LUNG FIBROSIS AT THE TIME WE 2274 01:22:58,343 --> 01:23:00,645 INITIATED THIS STUDY. 2275 01:23:00,645 --> 01:23:02,714 OUR IMAGEERS GOT AFTER IT WITH 2276 01:23:02,714 --> 01:23:06,351 ADULT COLLEAGUES TO CREATE AN 2277 01:23:06,351 --> 01:23:08,086 IMAGING DIAGNOSIS OF FIBROSIS 2278 01:23:08,086 --> 01:23:10,789 WORKING WITH ADULT IMAGEERS, 2279 01:23:10,789 --> 01:23:15,127 LOOKING AT ADULT STUDIES AND 2280 01:23:15,127 --> 01:23:17,362 MAKING A DIAGNOSIS OF PEDIATRIC 2281 01:23:17,362 --> 01:23:19,131 LUNG FIBROSIS NOT IN THE 2282 01:23:19,131 --> 01:23:19,664 LITERATURE. 2283 01:23:19,664 --> 01:23:22,300 SO THIS IS ONE OF THE FIRST 2284 01:23:22,300 --> 01:23:26,037 BARR HAD TO ANDMEALLY WE DEVEL 2285 01:23:26,037 --> 01:23:28,273 CHANGED THE FIELD BECAUSE NOW 2286 01:23:28,273 --> 01:23:30,809 PEDIATRIC PULMONOLOGISTS ARE 2287 01:23:30,809 --> 01:23:32,344 LOOKING TO RADIOLOGISTS TO MAKE 2288 01:23:32,344 --> 01:23:35,647 THIS CALL ON KIDS WITH PULMONARY 2289 01:23:35,647 --> 01:23:35,914 FIBROSIS. 2290 01:23:35,914 --> 01:23:37,549 THIS WAS A BIG BARRIER WE 2291 01:23:37,549 --> 01:23:41,887 OVERCAME IN OUR DESIGN. 2292 01:23:41,887 --> 01:23:42,087 NEXT. 2293 01:23:42,087 --> 01:23:44,289 AGAIN, LUNG BIOPSIES ARE THE 2294 01:23:44,289 --> 01:23:46,358 GOLD STANDARD FOR FIBROSIS. 2295 01:23:46,358 --> 01:23:48,560 YOU CAN SEE CLASSIC LUNG 2296 01:23:48,560 --> 01:23:50,362 FIBROSIS STUDIES HERE, BUT AS I 2297 01:23:50,362 --> 01:23:52,831 SAID AND AS YOU HAVE HEARD, AS 2298 01:23:52,831 --> 01:24:00,138 MORE AND MORE JEANEGENETIC DISE 2299 01:24:00,138 --> 01:24:02,073 DOESN'T ALLOW US TO USE GOLD 2300 01:24:02,073 --> 01:24:05,177 STANDARD DIAGNOSIS FOR FIBROSIS. 2301 01:24:05,177 --> 01:24:12,284 SO CT SCAN OR INCLUSION BECAME 2302 01:24:12,284 --> 01:24:13,985 CRITICALLY IMPORTANT. 2303 01:24:13,985 --> 01:24:16,087 WE ARE BEGINNING TO GRAPPLE WITH 2304 01:24:16,087 --> 01:24:17,989 HOW DOES THIS LOOK DIFFEN 2305 01:24:17,989 --> 01:24:19,057 ADULTS AND PEDIATRICS AND 2306 01:24:19,057 --> 01:24:20,926 LOOKING FOR KIDS TO BRING TRIALS 2307 01:24:20,926 --> 01:24:22,594 FORWARD WITH, THERE IS A 2308 01:24:22,594 --> 01:24:23,261 DEVELOPMENTAL PROCESS, THERE IS 2309 01:24:23,261 --> 01:24:25,230 GROWTH IN THE LUNG, THERE ARE 2310 01:24:25,230 --> 01:24:26,198 CHANGES OVER TIME SO THOSE HAVE 2311 01:24:26,198 --> 01:24:28,133 TO BE INCLUDED. 2312 01:24:28,133 --> 01:24:30,435 AS WE THINK ABOUT IT, THERE HAS 2313 01:24:30,435 --> 01:24:35,740 TO BE MORE PATHOPHYSIOLOGIC 2314 01:24:35,740 --> 01:24:37,442 COMPARISONS TO IMAGING. 2315 01:24:37,442 --> 01:24:39,077 THAT WORK STILL NEEDS TO BE 2316 01:24:39,077 --> 01:24:41,246 DONE, BUT WE WERE STILL ABLE TO 2317 01:24:41,246 --> 01:24:51,590 MOVE FORWARD WITH THIS STUDY IN. 2318 01:24:51,590 --> 01:24:51,790 NEXT. 2319 01:24:51,790 --> 01:24:53,592 SO THIS STUDY WAS CONDUCTED, 2320 01:24:53,592 --> 01:24:56,294 THIS WAS A WORLDWIDE GLOBAL 2321 01:24:56,294 --> 01:24:56,495 STUDY. 2322 01:24:56,495 --> 01:24:57,696 VERY PROUD OF OUR COMMUNITY. 2323 01:24:57,696 --> 01:25:00,265 IT WAS CONDUCTED IN 53 LOCATIONS 2324 01:25:00,265 --> 01:25:01,800 IN 22 COUNTRIES. 2325 01:25:01,800 --> 01:25:05,437 THIS WAS A REAL TRIBUTE TO OUR 2326 01:25:05,437 --> 01:25:08,807 COMMUNITY AROUND THE WORLD 2327 01:25:08,807 --> 01:25:11,476 BANDING TOGETHER TO THINK ABOUT 2328 01:25:11,476 --> 01:25:13,311 KIDS WITH RARE DISEASE, IN A 2329 01:25:13,311 --> 01:25:14,880 COMMUNITY THAT NEVER CONDUCTED A 2330 01:25:14,880 --> 01:25:15,080 TRIAL. 2331 01:25:15,080 --> 01:25:17,415 I'M VERY PROUD OF THIS TRIAL IN 2332 01:25:17,415 --> 01:25:19,518 THAT TRIAL WAS CONDUCTED DURING 2333 01:25:19,518 --> 01:25:19,718 COVID. 2334 01:25:19,718 --> 01:25:21,486 IT HAD TO START AND RESTART. 2335 01:25:21,486 --> 01:25:23,154 THERE WERE SOME BARRIERS GETTING 2336 01:25:23,154 --> 01:25:26,625 THIS TRIAL OFF THE GROUND. 2337 01:25:26,625 --> 01:25:26,925 NEXT. 2338 01:25:26,925 --> 01:25:32,697 THE DESIGN OF THIS STUDY WAS 2339 01:25:32,697 --> 01:25:37,969 REALLY A TWO TO ONE NINTEDANIB 2340 01:25:37,969 --> 01:25:46,845 PLA SEENO, DOUBLE BLIND PLACEBO 2341 01:25:46,845 --> 01:25:47,078 CONTROL. 2342 01:25:47,078 --> 01:25:49,080 THE REST OF THE TRIAL IS 52 2343 01:25:49,080 --> 01:25:51,850 WEEKS SO THIS DESIGN WAS FAIRLY 2344 01:25:51,850 --> 01:25:52,083 CLASSIC. 2345 01:25:52,083 --> 01:25:55,587 MOST OF THE DATA WE EVALUATED 2346 01:25:55,587 --> 01:25:57,989 AROUND EFFICACY AND SITE EFFECT 2347 01:25:57,989 --> 01:26:00,058 PROFILE CAME FROM THE 20 WEEKS 2348 01:26:00,058 --> 01:26:01,760 OF THE DOUBLE BLIND PERIOD. 2349 01:26:01,760 --> 01:26:01,960 NEXT. 2350 01:26:01,960 --> 01:26:04,663 SO OUR OUTCOME MEASURES WERE 2351 01:26:04,663 --> 01:26:07,232 PRTLY PRIMARILY PK AND 2352 01:26:07,232 --> 01:26:07,699 SAFETY. 2353 01:26:07,699 --> 01:26:09,401 WE DID LOOK AT OTHER STUDY 2354 01:26:09,401 --> 01:26:13,972 MEASURES THAT WERE EXPLORED. 2355 01:26:13,972 --> 01:26:16,641 EFFICACY RELATED TO PULMONARY 2356 01:26:16,641 --> 01:26:16,908 FUNCTION. 2357 01:26:16,908 --> 01:26:19,711 THIS STUDY WAS NOT POWERED FOR 2358 01:26:19,711 --> 01:26:22,447 ANY OF THESE OTHER IMPACTS. 2359 01:26:22,447 --> 01:26:22,914 EXPLORE 2360 01:26:22,914 --> 01:26:28,753 CT SCANS, 2361 01:26:28,753 --> 01:26:30,188 EXPLORATORY BIOMARKERS. 2362 01:26:30,188 --> 01:26:33,992 WE HAVE PUBLISHED OND THIS 2363 01:26:33,992 --> 01:26:40,231 RELATED TO HRCT IS COMING 2364 01:26:40,231 --> 01:26:42,434 WE HAVE A BIOMARKER STUDY COMING 2365 01:26:42,434 --> 01:26:44,836 AND WE'VE HAD THIS IN ABSTRACT 2366 01:26:44,836 --> 01:26:45,203 FORM. 2367 01:26:45,203 --> 01:26:45,437 NEXT. 2368 01:26:45,437 --> 01:26:47,339 SO THIS WAS THE WAY THE DOUBLE 2369 01:26:47,339 --> 01:26:50,675 BLIND PLACEBO GROUPS WERE 2370 01:26:50,675 --> 01:26:50,909 ENROLL. 2371 01:26:50,909 --> 01:26:54,179 WE HAD 8' PATIENTS SCREENED. 2372 01:26:54,179 --> 01:26:57,282 WE HAD A CLINICAL OVERSITE 2373 01:26:57,282 --> 01:26:59,117 COMMITTEE THAT REVIEWED CRITERIA 2374 01:26:59,117 --> 01:27:00,819 FOR ENROLLMENT. 2375 01:27:00,819 --> 01:27:03,321 OUR COMMUNITY HAD NOT DONE THESE 2376 01:27:03,321 --> 01:27:03,922 STUDIES BEFORE. 2377 01:27:03,922 --> 01:27:06,391 WE WERE DEALING WITH NEW 2378 01:27:06,391 --> 01:27:08,493 DEFINITIONS FOR FIBROSIS AND THE 2379 01:27:08,493 --> 01:27:09,961 MAJORITY OF THESE PATIENTS THAT 2380 01:27:09,961 --> 01:27:13,131 WERE NOT INCLUDED, WERE NOT 2381 01:27:13,131 --> 01:27:15,567 INCLUDED BECAUSE PATIENTS WERE 2382 01:27:15,567 --> 01:27:22,107 TRYING TO ENROLL PATIENTS WITH 2383 01:27:22,107 --> 01:27:24,643 BRONCULITIS. 2384 01:27:24,643 --> 01:27:28,146 35 WERE RANDOMIZED TOOPLACEBO. 2385 01:27:28,146 --> 01:27:29,648 THE MAJORITY OF PATIENTS DID 2386 01:27:29,648 --> 01:27:30,215 COMPLETE THIS TRIAL. 2387 01:27:30,215 --> 01:27:37,656 NEXT. 2388 01:27:37,656 --> 01:27:40,225 THE DIAGNOSES ENROLLED, THE 2389 01:27:40,225 --> 01:27:44,829 SURFACTANT PROTEIN DEFICIENCY, 2390 01:27:44,829 --> 01:27:46,264 THE MOST COMMON DEFINITION IN 2391 01:27:46,264 --> 01:27:48,967 PATIENTS THAT WERE ENROLLED,RO 2392 01:27:48,967 --> 01:27:51,603 SYSTEMICSY SCLEROSIS PATIENTS WE 2393 01:27:51,603 --> 01:27:54,539 ENBUT OF NOTE, THE 2394 01:27:54,539 --> 01:27:56,675 LARGEST GROUP OF PATIENTS WITH 2395 01:27:56,675 --> 01:27:58,910 DIAGNOSE OF ONE WITH FIBROSIS. 2396 01:27:58,910 --> 01:28:01,646 MANY PATIENTS HAVE PULMONARY 2397 01:28:01,646 --> 01:28:01,946 FIBROSIS. 2398 01:28:01,946 --> 01:28:05,583 SO THIS IS A BASKET APPROACH TO 2399 01:28:05,583 --> 01:28:08,953 KIDS WITH PULMONARY FIBROSIS. 2400 01:28:08,953 --> 01:28:09,187 NEXT. 2401 01:28:09,187 --> 01:28:12,357 SO AT THE END OF THE DAY I'M NOT 2402 01:28:12,357 --> 01:28:13,324 GOING TO TAKE YOU THROUGH ALL 2403 01:28:13,324 --> 01:28:15,727 THE STUDY, BUT I WILL TAKE YOU 2404 01:28:15,727 --> 01:28:19,297 TO THE STUDY. 2405 01:28:19,297 --> 01:28:21,032 NINTEDANIB HAD AN ACCEPTABLE 2406 01:28:21,032 --> 01:28:22,367 SAFETY AND TOLL REHABILITATE 2407 01:28:22,367 --> 01:28:23,968 PROFILE WITH NO NEW SAFETY 2408 01:28:23,968 --> 01:28:25,904 SIGNALS COMPARED TO ADULTS. 2409 01:28:25,904 --> 01:28:27,305 THAT WAS A HUGE SUCCESS. 2410 01:28:27,305 --> 01:28:30,141 WE WERE ABLE TO DO WEIGHT-BASED 2411 01:28:30,141 --> 01:28:31,076 DOSING. 2412 01:28:31,076 --> 01:28:33,411 AS A PEDIATRICIAN WE HAVE TO DO 2413 01:28:33,411 --> 01:28:36,147 WEIGHT-BASED DOSING TO GET IN 2414 01:28:36,147 --> 01:28:45,156 THE SAME TREATMENT RANGES ANDM . 2415 01:28:45,156 --> 01:28:47,859 WE WERE ABLE TO DO WEIGHT-BASED 2416 01:28:47,859 --> 01:28:51,863 DOSING AND GET IN THE RIGHT 2417 01:28:51,863 --> 01:28:53,031 RANGES FOR ADULTS. 2418 01:28:53,031 --> 01:28:59,304 WE LOOKED AT EFFICACY MEASURES 2419 01:28:59,304 --> 01:28:59,771 TO TREND. 2420 01:28:59,771 --> 01:29:04,709 TRENDED IN FAVOR OF 2421 01:29:04,709 --> 01:29:05,744 NI THOSE THIS WAS NOT 2422 01:29:05,744 --> 01:29:08,279 POWERED FOR THAT. 2423 01:29:08,279 --> 01:29:11,483 WE DO HAVE AN INPEDILD ON STUDY 2424 01:29:11,483 --> 01:29:13,651 THAT ENROLLED NEW PATIENTS TO 2425 01:29:13,651 --> 01:29:19,124 LOOK AT ONGOING SAFETY. 2426 01:29:19,124 --> 01:29:20,892 OUR SCIENTIFIC COMMUNITY 2427 01:29:20,892 --> 01:29:23,595 BELIEVES THAT THIS DATA SUPPORTS 2428 01:29:23,595 --> 01:29:24,629 POSITIVE BENEFIT RISK ASSESSMENT 2429 01:29:24,629 --> 01:29:28,833 FOR THE USE OF NINTEDANIB FOR 2430 01:29:28,833 --> 01:29:34,205 USE IN CHILDREN WITH DOSING 2431 01:29:34,205 --> 01:29:35,707 INPED ILB. 2432 01:29:35,707 --> 01:29:36,174 NEXT. 2433 01:29:36,174 --> 01:29:42,213 I WILL FLENGS HAVE -- MENTION 2434 01:29:42,213 --> 01:29:46,818 THERE HAVE BEEN OTHER PAPERS 2435 01:29:46,818 --> 01:29:47,352 PUBLISHED. 2436 01:29:47,352 --> 01:29:51,122 THESE AYSEIAN DYNAMIC APPROACHES 2437 01:29:51,122 --> 01:29:54,859 TO TAKE A SMALL POPULATION, OUR 2438 01:29:54,859 --> 01:29:56,628 GROUP, ADD IN DATA FROM THE 2439 01:29:56,628 --> 01:29:59,297 ADULT STUDIES, LOOKING AT THOSE 2440 01:29:59,297 --> 01:30:02,600 ADULT STUDIES, META ANALYSIS OF 2441 01:30:02,600 --> 01:30:04,736 THOSE ADULT TRIALS AND THEN ADD 2442 01:30:04,736 --> 01:30:08,106 THEM TO OUR STUDY, OUR DATA FROM 2443 01:30:08,106 --> 01:30:10,308 THE PEDIATRIC TRIAL AND COME UP 2444 01:30:10,308 --> 01:30:12,277 WITH SOME IDEA OF EFFICACY. 2445 01:30:12,277 --> 01:30:20,552 SO WE WERE ASKED TO DO THESE 2446 01:30:20,552 --> 01:30:20,952 BAY 2447 01:30:20,952 --> 01:30:21,753 BAYESIAN TRIALS. 2448 01:30:21,753 --> 01:30:25,790 WE HAD TO DO PRESPECIFIED 2449 01:30:25,790 --> 01:30:28,126 WEIGHTING OF HOW MUCH PEDIATRIC 2450 01:30:28,126 --> 01:30:30,295 ILD WAS LIKE ADULT ILD TO DO 2451 01:30:30,295 --> 01:30:31,362 THESE STUDIES. 2452 01:30:31,362 --> 01:30:33,364 AT THE END OF THE DAY WE WERE 2453 01:30:33,364 --> 01:30:37,135 ABLE TO LOOK AT OUR FINDINGS FOR 2454 01:30:37,135 --> 01:30:40,505 PULMONARY FUNCTION AND COMBINE 2455 01:30:40,505 --> 01:30:42,173 DECIDE WHETHER THE EFFICACY NOW 2456 01:30:42,173 --> 01:30:44,509 WITH THE POPULATION THAT WAS 2457 01:30:44,509 --> 01:30:46,311 POWERED TO LOOK AT THIS WAS 2458 01:30:46,311 --> 01:30:46,945 STILL SUBSTANTIAL. 2459 01:30:46,945 --> 01:30:50,415 NEXT. 2460 01:30:50,415 --> 01:30:54,018 SO THIS IS FROM THE PAPER WE 2461 01:30:54,018 --> 01:31:04,929 EFESSEF OF NINTEDANILB 2462 01:31:06,397 --> 01:31:09,234 OVER THE DOUBLE BLIND PLACEBO 2463 01:31:09,234 --> 01:31:09,534 CONTROL. 2464 01:31:09,534 --> 01:31:14,405 THERE IS A 1.65 DIFFERENCE IN 2465 01:31:14,405 --> 01:31:19,477 THE NINTEDANIB GRUBE FROM 2466 01:31:19,477 --> 01:31:21,279 PLACEBO GROUP. 2467 01:31:21,279 --> 01:31:24,582 OUR TRIAL WAS 1.2 PERCENT 2468 01:31:24,582 --> 01:31:28,753 DIFFERENCE BETWEEN NINTEDANIB 2469 01:31:28,753 --> 01:31:30,088 AND THE PLACEBO. 2470 01:31:30,088 --> 01:31:34,559 WHEN YOU LOOK AT THE COMBINED 2471 01:31:34,559 --> 01:31:37,862 BAYESIAN ANALYSIS THERE IS 137B9 2472 01:31:37,862 --> 01:31:38,129 63. 2473 01:31:38,129 --> 01:31:40,865 THAT HAS BEEN POWERED TO LOOK AT 2474 01:31:40,865 --> 01:31:41,132 EFFICACY. 2475 01:31:41,132 --> 01:31:43,368 REQUIRED BY THE REGULATORY 2476 01:31:43,368 --> 01:31:45,536 AGENCIES TO EXPLORE THAT. 2477 01:31:45,536 --> 01:31:48,840 THESE BAYESIAN TECH TEAKS ARE 2478 01:31:48,840 --> 01:31:51,075 IMPORTANT WITH SMALL POPULATIONS 2479 01:31:51,075 --> 01:31:53,244 AND OUR DATA USING THESE 2480 01:31:53,244 --> 01:31:53,978 TECHNIQUES WAS SIGNIFICANT. 2481 01:31:53,978 --> 01:32:01,953 NEXT. 2482 01:32:01,953 --> 01:32:04,322 SO I WANT TO HIGHLIGHT THIS AS I 2483 01:32:04,322 --> 01:32:06,090 HAVE HAD TO SAY THIS MANY TIMES 2484 01:32:06,090 --> 01:32:08,660 BECAUSEES THINK IT IS SO T 2485 01:32:08,660 --> 01:32:08,927 IMPORTANT. 2486 01:32:08,927 --> 01:32:11,729 THE TENTATIVE IS THE MOST 2487 01:32:11,729 --> 01:32:16,968 STUDY -- NINTEDANIB IS THE MOST 2488 01:32:16,968 --> 01:32:18,603 STUDIED DRUG I CAN PRESCRIBE FOR 2489 01:32:18,603 --> 01:32:21,806 A CHILD WITH FIBROSIS.IS KNOWHE 2490 01:32:21,806 --> 01:32:26,511 THE DOSING. 2491 01:32:26,511 --> 01:32:29,380 I KNOW BASED ON STATISTICAL 2492 01:32:29,380 --> 01:32:31,582 ANALYSIS THERE APPEARS TO BE 2493 01:32:31,582 --> 01:32:32,383 EFFICACY AND IT IS THE MOST I 2494 01:32:32,383 --> 01:32:38,423 KNOW. 2495 01:32:38,423 --> 01:32:41,225 THE STUDY I SHOWED YOU ABOUT 2496 01:32:41,225 --> 01:32:43,294 WHON'T KNOW AND POTENTIAL 2497 01:32:43,294 --> 01:32:45,830 RISKS TO PATIENTS CAUSES ME 2498 01:32:45,830 --> 01:32:47,999 GREAT PAUSE. 2499 01:32:47,999 --> 01:32:54,072 THIS IS AN IMPORTANT STATE. 2500 01:32:54,072 --> 01:32:55,974 WHAT REGULATORY ACTION HAS 2501 01:32:55,974 --> 01:32:56,808 HAPPENED? 2502 01:32:56,808 --> 01:33:00,011 A BIG BREAK THROUGH OF PEDIATRIC 2503 01:33:00,011 --> 01:33:02,046 INVESTIGATIONAL PLANS ARE BEING 2504 01:33:02,046 --> 01:33:04,515 REQUIRED BY FDA AND EMA. 2505 01:33:04,515 --> 01:33:08,920 IT ALLOWED KIDS TO BE OPEN TO P. 2506 01:33:08,920 --> 01:33:09,120 NEXT. 2507 01:33:09,120 --> 01:33:09,988 SO THE FDA. 2508 01:33:09,988 --> 01:33:17,862 RESPONSE LETTER IN REGARD TO OUR 2509 01:33:17,862 --> 01:33:20,064 TREATMENT. 2510 01:33:20,064 --> 01:33:20,698 NEXT. 2511 01:33:20,698 --> 01:33:22,100 NEY FOUND THAT FDA COULD NOT 2512 01:33:22,100 --> 01:33:24,302 APPROVE THE APPLICATION IN ITS 2513 01:33:24,302 --> 01:33:25,136 CURRENT FORM FOR CHILDREN. 2514 01:33:25,136 --> 01:33:32,076 NEXT. 2515 01:33:32,076 --> 01:33:34,112 BUT THEY SAID THIS LETTER HAD NO 2516 01:33:34,112 --> 01:33:37,882 IMPACT ON OUR ABILITY TO 2517 01:33:37,882 --> 01:33:44,122 CONTINUE WITH THE INPED STUDY TO 2518 01:33:44,122 --> 01:33:46,357 LOOK AT NINTEDANIB IN CHILDREN 2519 01:33:46,357 --> 01:33:48,726 IN AN OPEN LABEL FASHION. 2520 01:33:48,726 --> 01:33:52,063 SO DID NOT GRANT 2521 01:33:52,063 --> 01:33:52,797 PEDIATRIC INDICATION FOR THE 2522 01:33:52,797 --> 01:33:56,367 MOST STUDIED DRUG OF KIDS COULD 2523 01:33:56,367 --> 01:33:58,236 I HAVE FOR PULMONARY FIBROSIS. 2524 01:33:58,236 --> 01:33:59,670 THERE IS BREAKING NEWS AS OF 2525 01:33:59,670 --> 01:34:00,772 LAST WEEK. 2526 01:34:00,772 --> 01:34:04,509 NEXT. 2527 01:34:04,509 --> 01:34:06,077 EMA, THE EUROPEAN MEDICAL 2528 01:34:06,077 --> 01:34:08,079 AGENCY, IF YOU ARE NOT FAMILIAR 2529 01:34:08,079 --> 01:34:10,648 WITH THIS IS SIMILAR TO THE FDA 2530 01:34:10,648 --> 01:34:11,516 IN EUROPE. 2531 01:34:11,516 --> 01:34:16,287 HAS NOW RECOMMENDED THE APPROVAL 2532 01:34:16,287 --> 01:34:18,689 OF NEW INDICATIONS AND THE 2533 01:34:18,689 --> 01:34:19,857 INDICATIONS OF EXTENSION OF 2534 01:34:19,857 --> 01:34:20,758 NINTEDANIB TO CHILDREN. 2535 01:34:20,758 --> 01:34:24,328 NEXT. 2536 01:34:24,328 --> 01:34:28,266 THEY RECOMME T APPROVAL FOR 2537 01:34:28,266 --> 01:34:30,902 OFEB FHILDREN FOR THE 2538 01:34:30,902 --> 01:34:32,203 TREATMENT OF CLINICALLY 2539 01:34:32,203 --> 01:34:34,839 SIGNIFICANT PROGRESSIVE 2540 01:34:34,839 --> 01:34:36,641 INTERSTITIAL LUNG DISEASE. 2541 01:34:36,641 --> 01:34:40,144 AND IN KIDS WITH SYSTEMIC 2542 01:34:40,144 --> 01:34:40,445 SCLEROSIS. 2543 01:34:40,445 --> 01:34:43,214 SO THEY GAVE TWO NEW LABELS 2544 01:34:43,214 --> 01:34:45,716 PROGRESSIVE FIBROSIS IN CHILDREN 2545 01:34:45,716 --> 01:34:50,521 6-17 YEARS OF AGE AND CHILDREN 2546 01:34:50,521 --> 01:34:52,623 6-17 WITH SYSTEMIC SCLEROSIS. 2547 01:34:52,623 --> 01:34:58,729 NEXT. WE ARE VERY PROUD OF THIS. 2548 01:34:58,729 --> 01:35:00,998 THIS WILL BECOME THE FIRST 2549 01:35:00,998 --> 01:35:02,366 APPROVED TREATMENT FOR CHILDREN 2550 01:35:02,366 --> 01:35:05,036 WITH FIBROSIS ABOVE THE AGE OF 6 2551 01:35:05,036 --> 01:35:06,804 AND FOR SYSTEMIC SCLEROSIS. 2552 01:35:06,804 --> 01:35:08,739 WE BELIEVE THIS HELPS VALIDATE 2553 01:35:08,739 --> 01:35:10,575 OUR CLINICAL DESIGN, OUR 2554 01:35:10,575 --> 01:35:12,176 ASSUMPTIONS AND THE WORK WE HAVE 2555 01:35:12,176 --> 01:35:14,679 DONE TO OVERCOME MANY RS, 2556 01:35:14,679 --> 01:35:16,147 BUT WE HAVE WORK TO DO 2557 01:35:16,147 --> 01:35:19,484 WITH FDA AND REGULATORY IN THE 2558 01:35:19,484 --> 01:35:21,519 U.S. ABOUT HOW TO GET DRUGS 2559 01:35:21,519 --> 01:35:21,752 FORWARD. 2560 01:35:21,752 --> 01:35:23,488 AND HOW TO THINK ABOUT THIS IN 2561 01:35:23,488 --> 01:35:24,055 NEW WAYS. 2562 01:35:24,055 --> 01:35:26,124 SO THIS IS A BARRIER WE ARE 2563 01:35:26,124 --> 01:35:26,858 GOING TO BE APPROACHING. 2564 01:35:26,858 --> 01:35:29,527 NEXT. 2565 01:35:29,527 --> 01:35:32,230 SO THE NINTEDANIB CLINICAL TRIAL 2566 01:35:32,230 --> 01:35:35,433 HAS HAD TREMENDOUS IMPACT ON 2567 01:35:35,433 --> 01:35:37,435 PEDIATRIC PULMONARY FIBROSIS. 2568 01:35:37,435 --> 01:35:41,239 I CAN'T TELL YOU HOW IT 2569 01:35:41,239 --> 01:35:42,440 JETSONNED THE FIELD FORWARD. 2570 01:35:42,440 --> 01:35:44,308 WE DON'T HAVE RESOURCES. 2571 01:35:44,308 --> 01:35:45,877 IT SHOWED OUR COMMUNITY CAN DO 2572 01:35:45,877 --> 01:35:46,177 THIS. 2573 01:35:46,177 --> 01:35:48,045 WE CAN DO IT WORLDWIDE. 2574 01:35:48,045 --> 01:35:51,048 I'M VERY PROUD OF OUR COMMUNITY. 2575 01:35:51,048 --> 01:35:53,951 I CONSIDER REALLY A GRAND SLAM. 2576 01:35:53,951 --> 01:35:56,454 WE HAD MAJOR ADVANCES ACHIEVED 2577 01:35:56,454 --> 01:35:57,688 DURING THIS STUDY. 2578 01:35:57,688 --> 01:36:00,191 NEW DEFINITIONS OF PULMONARY 2579 01:36:00,191 --> 01:36:03,227 FIBROSIS IN IMAGING WERE ARRIVED 2580 01:36:03,227 --> 01:36:03,761 AT. 2581 01:36:03,761 --> 01:36:10,301 PRIOR TO THIS STUDY PEDIATRIC 2582 01:36:10,301 --> 01:36:13,004 RADIOLOGISTS WERE TOLD TO NOT 2583 01:36:13,004 --> 01:36:14,739 COMMENT ON FIBROSIS. 2584 01:36:14,739 --> 01:36:18,476 NOW THIS DEFINITION IS 2585 01:36:18,476 --> 01:36:20,077 FORTHCOMING PEDIATRIC 2586 01:36:20,077 --> 01:36:21,212 RADIOLOGISTS ARE BEING TOLD 2587 01:36:21,212 --> 01:36:23,748 ABOUT THE DIAGNOSIS AND THE 2588 01:36:23,748 --> 01:36:26,817 INDICATIONS AND CRITERIA FOR 2589 01:36:26,817 --> 01:36:32,456 FIBROSIS AND THOSE CTs ARE READ 2590 01:36:32,456 --> 01:36:35,927 AS FIBROSIS THAT GETS YOU TO THE 2591 01:36:35,927 --> 01:36:37,161 PULMONOLOGIST FAST. 2592 01:36:37,161 --> 01:36:38,696 IT IS MORE COMMON THAN YOU THINK 2593 01:36:38,696 --> 01:36:40,565 SO THIS IS A MAJOR ACHIEVEMENT 2594 01:36:40,565 --> 01:36:44,335 TO HAVE A DIAGNOSIS WITH CT THAT 2595 01:36:44,335 --> 01:36:46,637 CAN BE EDUCATED THROUGH THE 2596 01:36:46,637 --> 01:36:47,872 PEDIATRIC IMAGING COMMUNITY. 2597 01:36:47,872 --> 01:36:49,740 NEW UNDERSTANDING HAS BEEN 2598 01:36:49,740 --> 01:36:49,974 GAINED. 2599 01:36:49,974 --> 01:36:52,210 WE HAVE MAJOR WORK TO DO WITH 2600 01:36:52,210 --> 01:36:54,178 REGULATORY ORGANIZATIONS 2601 01:36:54,178 --> 01:36:55,846 INCLUDING PATIENT ADVOCACY 2602 01:36:55,846 --> 01:36:59,016 GROUPS AND THE PEDIATRIC 2603 01:36:59,016 --> 01:37:01,385 PULMONARY IMAGING AND ADULT 2604 01:37:01,385 --> 01:37:01,686 COMMUNITIES. 2605 01:37:01,686 --> 01:37:05,456 THERE IS A LARGE PIPELINE OF 2606 01:37:05,456 --> 01:37:06,490 MEDICATIONS FORTHCOMING THAT 2607 01:37:06,490 --> 01:37:07,258 COULD HELP CHILDREN. 2608 01:37:07,258 --> 01:37:09,527 SOME DESIGNS COULD BE HELPFUL 2609 01:37:09,527 --> 01:37:13,297 AND VALIDATED SOME OF THOSE 2610 01:37:13,297 --> 01:37:14,899 ASSUMPTIONS VALIDATED IN EUROPE. 2611 01:37:14,899 --> 01:37:17,134 WE HAVE MAJOR WORK TO DO TO 2612 01:37:17,134 --> 01:37:18,169 THINK ABOUT DIFFERENCES IN ADULT 2613 01:37:18,169 --> 01:37:19,270 AND PEDIATRICS. 2614 01:37:19,270 --> 01:37:23,874 SO SOME OF THESE BAYESIAN 2615 01:37:23,874 --> 01:37:25,142 ANALYSIS, PEOPLE CAN FEEL 2616 01:37:25,142 --> 01:37:27,144 COMFORTABLE WE ARE COMPARING THE 2617 01:37:27,144 --> 01:37:27,812 RIGHT GROUPS. 2618 01:37:27,812 --> 01:37:30,548 I SAID IN MY SLIDE THE FIRST 2619 01:37:30,548 --> 01:37:31,382 PEDIATRIC INDICATION QUESTION 2620 01:37:31,382 --> 01:37:32,984 MARK BECAUSE THAT WAS BREAKING 2621 01:37:32,984 --> 01:37:34,285 NEWS LAST WEEK AND NOW A WE HAV 2622 01:37:34,285 --> 01:37:34,485 THAT. 2623 01:37:34,485 --> 01:37:37,321 NEXT. 2624 01:37:37,321 --> 01:37:39,457 SO I WANT TO THANK THE CHILD 2625 01:37:39,457 --> 01:37:40,424 FOUNDATION AND ALL THE FAMILIES 2626 01:37:40,424 --> 01:37:42,660 THAT ARE A PART OF OUR RESEARCH 2627 01:37:42,660 --> 01:37:44,095 NETWORK THAT HELP US THINK ABOUT 2628 01:37:44,095 --> 01:37:45,730 THIS EVERY DAY. 2629 01:37:45,730 --> 01:37:48,366 THERE WAS ASOUS AMOUNT OF 2630 01:37:48,366 --> 01:37:48,699 COLLABORATION. 2631 01:37:48,699 --> 01:37:57,241 I WANT TO THANK 2632 01:37:57,241 --> 01:37:57,975 BE-INGELHEIM FORG 2633 01:37:57,975 --> 01:37:58,976 FAITH IN US. 2634 01:37:58,976 --> 01:38:00,311 WITH THAT, I WILL END. 2635 01:38:00,311 --> 01:38:01,379 >> THANK YOU, ROBIN. 2636 01:38:01,379 --> 01:38:04,282 >> FINALLY, WE WILL TURN TO 2637 01:38:04,282 --> 01:38:05,449 KAREN SCHNEIDER WHO IS GOING TO 2638 01:38:05,449 --> 01:38:07,151 HELP TALKLP US ABOUTOME 2639 01:38:07,151 --> 01:38:08,686 THINGS THAT ARE MAYBE THE 2640 01:38:08,686 --> 01:38:10,821 CLINICIAN RESEARCHERS OF US MAY 2641 01:38:10,821 --> 01:38:12,823 KIND OF, I DON'T KNOW, DRAW AWAY 2642 01:38:12,823 --> 01:38:14,925 FROM THIS TOPIC. 2643 01:38:14,925 --> 01:38:16,794 SO I'M GLAD WE HAVE SOMEBODY TO 2644 01:38:16,794 --> 01:38:20,131 CHAT MORE AND HIP US THINK ABOUT 2645 01:38:20,131 --> 01:38:22,066 THIS PART OF THINGS. 2646 01:38:22,066 --> 01:38:23,000 THANK YOU, KAREN. 2647 01:38:23,000 --> 01:38:24,302 >> YES, KATELYN. 2648 01:38:24,302 --> 01:38:25,670 CAN YOU HEAR ME? 2649 01:38:25,670 --> 01:38:26,737 OKAY. 2650 01:38:26,737 --> 01:38:27,104 GREAT. 2651 01:38:27,104 --> 01:38:27,305 YEAH. 2652 01:38:27,305 --> 01:38:32,643 AS KATELYN SAID, I HAVE BEEN ANT 2653 01:38:32,643 --> 01:38:34,378 ASR ASSO. 2654 01:38:34,378 --> 01:38:37,581 BEFORE THAT I WAS -- I DID WORK 2655 01:38:37,581 --> 01:38:40,151 AT UNIVERSITY TECH TRANSFER 2656 01:38:40,151 --> 01:38:43,020 DURING LAW SCHOOL AND BEFORE 2657 01:38:43,020 --> 01:38:46,057 THAT I WAS A LAB SCIENTIST. 2658 01:38:46,057 --> 01:38:51,128 I'M PARTICULA SYMPATHETIC TO SG 2659 01:38:51,128 --> 01:38:53,764 PROCESS CAN CAUSE. 2660 01:38:53,764 --> 01:38:57,301 I MAKE EVERYONE WHO SENDS MY 2661 01:38:57,301 --> 01:38:59,136 SLIDES PUT IN THIS DISCLAIMER, 2662 01:38:59,136 --> 01:39:00,871 MY STATEMENTS AND OPINIONS ARE 2663 01:39:00,871 --> 01:39:06,344 MY OWN AND MAY NOT REFLECT MY 2664 01:39:06,344 --> 01:39:06,644 EMPLOYER. 2665 01:39:06,644 --> 01:39:08,012 I'M GOING TO TALK SPECIFICALLY 2666 01:39:08,012 --> 01:39:11,015 ABOUT INVESTIGATOR-INITIATED 2667 01:39:11,015 --> 01:39:13,184 CLINICAL TRIAL CONTRACTING. 2668 01:39:13,184 --> 01:39:18,022 IF THERE ARE QUESTIONS ABOUT 2669 01:39:18,022 --> 01:39:19,957 CLINICAL TRIAL CONTRACTING 2670 01:39:19,957 --> 01:39:21,926 GENERALLY, THAT COULD BE FOR THE 2671 01:39:21,926 --> 01:39:22,093 Q&A. 2672 01:39:22,093 --> 01:39:24,962 WE ARE TALKING ABOUT CLINICAL 2673 01:39:24,962 --> 01:39:26,931 TRIALS DEVELOPED DESIGNED AND 2674 01:39:26,931 --> 01:39:28,933 CONDUCTED WITH AN INVESTIGATOR 2675 01:39:28,933 --> 01:39:29,767 WITHOUT PARTICIPATION OTHER THAN 2676 01:39:29,767 --> 01:39:33,304 CERTAIN REVIEW AND APPROVAL 2677 01:39:33,304 --> 01:39:35,873 RATES OF THE COMPANY SUPPORTER. 2678 01:39:35,873 --> 01:39:38,209 THE INVESTIGATOR CAN REQUEST 2679 01:39:38,209 --> 01:39:42,713 DRUG FUNDING OR BOTH FROM 2680 01:39:42,713 --> 01:39:43,748 PHARMACEUTICAL COMPANY. 2681 01:39:43,748 --> 01:39:46,350 OR FUNDING FOR A 2682 01:39:46,350 --> 01:39:47,218 NONINTERVENTIONAL STUDY. 2683 01:39:47,218 --> 01:39:49,320 I'M GOING TO TALK MORE ABOUT 2684 01:39:49,320 --> 01:39:51,422 INTERVENTIONAL STUDIES. 2685 01:39:51,422 --> 01:39:54,058 THE INVESTIGATOR IS THE 2686 01:39:54,058 --> 01:39:55,526 REGULATORY SPONS AND FULFILLS 2687 01:39:55,526 --> 01:39:58,596 ALL OF THOSE OBLIGATIONS AS SET 2688 01:39:58,596 --> 01:40:01,065 FORTH IN THE CLINICAL TRIAL 2689 01:40:01,065 --> 01:40:01,465 REGULATIONS. 2690 01:40:01,465 --> 01:40:03,734 RESPONSIBLE FOR ALL OF THE 2691 01:40:03,734 --> 01:40:05,770 CONDUCT MONITORING SAFETY 2692 01:40:05,770 --> 01:40:07,171 RECORDING DATA ANALYSIS 2693 01:40:07,171 --> 01:40:09,940 OBLIGATIONS OF ANY STUDY 2694 01:40:09,940 --> 01:40:10,574 SPONSOR. 2695 01:40:10,574 --> 01:40:13,744 TYPICALLY A DRUG COMPANY WILL 2696 01:40:13,744 --> 01:40:16,113 HAVE ON ITS WEBSITE INSTRUCTIONS 2697 01:40:16,113 --> 01:40:18,616 FOR SUBMITTING A REQUEST FOR 2698 01:40:18,616 --> 01:40:20,985 SUPPORT AND THERE WILL BE 2699 01:40:20,985 --> 01:40:22,319 INFORMATION ABOUT THE COMPANY'S 2700 01:40:22,319 --> 01:40:26,724 RESEARCH AREAS O INTEREST. 2701 01:40:26,724 --> 01:40:28,259 BECAUSE THE COMPANY IS GOING TO 2702 01:40:28,259 --> 01:40:30,528 SUPPORT STUDIES THAT ALIGN WITH 2703 01:40:30,528 --> 01:40:34,298 ITS OWN RESEARCH INTERESTS. 2704 01:40:34,298 --> 01:40:36,367 MIGHT HELP INFORM THE USE OF THE 2705 01:40:36,367 --> 01:40:38,636 DRUG AND ALSO ADDRESS AN UNMET 2706 01:40:38,636 --> 01:40:39,303 MEDICAL NEED. 2707 01:40:39,303 --> 01:40:42,940 GO TO THE NEXT SLIDE. 2708 01:40:42,940 --> 01:40:48,312 WE HEA A LOT ABOUT THE BARRIERS 2709 01:40:48,312 --> 01:40:49,747 THAT EXIST WITHIN CONTRACTING. 2710 01:40:49,747 --> 01:40:51,482 MYRA ONE TIP IS ALWAYS GET 2711 01:40:51,482 --> 01:40:53,250 ON THE PHONE. 2712 01:40:53,250 --> 01:40:55,252 I COULD PROBABLY TALK FOR 20 2713 01:40:55,252 --> 01:40:56,687 MINUTES ABOUT THE FRUSTRATIONS. 2714 01:40:56,687 --> 01:40:58,989 THESE DAYS IT IS EASY TO GO BACK 2715 01:40:58,989 --> 01:41:00,724 AND FORTH IN COMMENTS AND THE 2716 01:41:00,724 --> 01:41:02,593 WORD DOCUMENT AND NEGOTIATE THAT 2717 01:41:02,593 --> 01:41:02,760 WAY. 2718 01:41:02,760 --> 01:41:04,161 THAT WILL TAKE MORE TIME THAN 2719 01:41:04,161 --> 01:41:05,963 GETTING ON THE PHONE. 2720 01:41:05,963 --> 01:41:07,765 I ALWAYS RECOMMEND THE 2721 01:41:07,765 --> 01:41:08,966 INVESTIGATOR JOINS THE CALL. 2722 01:41:08,966 --> 01:41:11,902 THAT WAY THE PARTIES ARE ALIGNED 2723 01:41:11,902 --> 01:41:14,271 AND YOU DON'T NEED A SECOND 2724 01:41:14,271 --> 01:41:14,472 CALL. 2725 01:41:14,472 --> 01:41:16,507 THE CONTRACT IS INTENDED TO 2726 01:41:16,507 --> 01:41:19,176 PROTECT BOTH PARTIES, DEFINE THE 2727 01:41:19,176 --> 01:41:20,945 ROLES AND RESPONSIBILITIES OF 2728 01:41:20,945 --> 01:41:21,412 EACH PARTY. 2729 01:41:21,412 --> 01:41:24,582 THAT INCLUDES THE CONDUCT OF THE 2730 01:41:24,582 --> 01:41:26,484 STUDY, THE SAFETY REPORTING 2731 01:41:26,484 --> 01:41:27,651 OBLIGATIONS, ANY REVIEW RIGHTS 2732 01:41:27,651 --> 01:41:28,652 THAT THE COMPANY MIGHT HAVE. 2733 01:41:28,652 --> 01:41:31,922 SO THAT IS USUALLY LIKE A 2734 01:41:31,922 --> 01:41:33,824 MONITORING PLAN OR SAFETY 2735 01:41:33,824 --> 01:41:34,658 MANAGEMENT PLAN. 2736 01:41:34,658 --> 01:41:36,660 ANY SORT OF PROTOCOL AMENDMENTS 2737 01:41:36,660 --> 01:41:37,995 THE COMPANY IS GOING TO REVIEW 2738 01:41:37,995 --> 01:41:40,731 THOSE IF THEY ARE SUPPORTING THE 2739 01:41:40,731 --> 01:41:40,931 STUDY. 2740 01:41:40,931 --> 01:41:43,467 THE CONTRACTEDDRESSES DRUG 2741 01:41:43,467 --> 01:41:43,701 SUPPLY. 2742 01:41:43,701 --> 01:41:46,637 HOW PAYMENT IS STRUCTURED, WHICH 2743 01:41:46,637 --> 01:41:48,472 WE'LL TALK ABOUT A LITTLE BIT 2744 01:41:48,472 --> 01:41:49,139 LATER. 2745 01:41:49,139 --> 01:41:50,875 BUT IT IS IMPORTANT THAT BOTH 2746 01:41:50,875 --> 01:41:52,076 PARTIES WHO ARE NEGOTIATING 2747 01:41:52,076 --> 01:41:54,812 UNDERSTAND THE GOALS FOR THE 2748 01:41:54,812 --> 01:41:55,179 STUDY. 2749 01:41:55,179 --> 01:41:56,514 I THINK FOR INVESTIGATOR 2750 01:41:56,514 --> 01:41:59,717 INITIATED TRIALS THE BIGGEST 2751 01:41:59,717 --> 01:42:02,553 ISSUE IS THAT THEY ARE GENERALLY 2752 01:42:02,553 --> 01:42:03,687 LESS COMMON THAN COMPANY 2753 01:42:03,687 --> 01:42:06,156 SPONSORED TRIALS. 2754 01:42:06,156 --> 01:42:09,226 IF A NEGOTIATOR ON EITHER SIDE 2755 01:42:09,226 --> 01:42:10,361 DUNCE UNDERSTAND THE DIFFERENCE 2756 01:42:10,361 --> 01:42:11,762 IN WHAT WE LOOK FOR THAT CAN 2757 01:42:11,762 --> 01:42:16,433 CAUSE DELAYS. 2758 01:42:16,433 --> 01:42:19,870 I HAVE PROBABLY DONE ABOUT 100 2759 01:42:19,870 --> 01:42:21,839 INVESTIGATOR INITIATED CONTRACTS 2760 01:42:21,839 --> 01:42:23,641 VERSUS THOUSANDS AND THOUSANDS 2761 01:42:23,641 --> 01:42:26,076 OF COMPANY SPONSORED CONTRACTS. 2762 01:42:26,076 --> 01:42:28,546 THE DRUG COMPANY PROVIDES THE 2763 01:42:28,546 --> 01:42:30,915 TEMPLATE 80% OF THE TIME. 2764 01:42:30,915 --> 01:42:33,651 THAT TEMPLATE ALLOWS FOR 2765 01:42:33,651 --> 01:42:37,121 FLEXIBILITY FOR DIFFERENT TYPES 2766 01:42:37,121 --> 01:42:40,324 OF STUDY STRUCTURES, IT IS GOOD 2767 01:42:40,324 --> 01:42:42,226 TO PLAN AHEAD FOR A TEMPLATE 2768 01:42:42,226 --> 01:42:45,162 THAT ALLOWS YOU TO FIT THE STUDY 2769 01:42:45,162 --> 01:42:49,767 YOU ARE NEGOTIATING FOR. 2770 01:42:49,767 --> 01:42:50,801 SO WHAT WE HEAR FROM 2771 01:42:50,801 --> 01:42:52,636 INVESTIGATORS IS THAT THE FOCUS 2772 01:42:52,636 --> 01:42:55,039 REALLY IS ON PATIENT CARE AND 2773 01:42:55,039 --> 01:42:57,107 SCIENTIFIC ADVANCEMENT. 2774 01:42:57,107 --> 01:43:01,312 THERE IS AN INTEREST FOR BOTH 2775 01:43:01,312 --> 01:43:02,479 SIDES IN THE INVESTIGATOR 2776 01:43:02,479 --> 01:43:04,148 PUBLISHING THE STUDY. 2777 01:43:04,148 --> 01:43:06,784 WHERE WE GET STUCK WHICH I WILL 2778 01:43:06,784 --> 01:43:08,586 TALK ABOUT ON THE NEXT SLIDE IS 2779 01:43:08,586 --> 01:43:10,821 THE AREAS OF FOCUS ON 2780 01:43:10,821 --> 01:43:12,523 NEGOTIATION COME DOWN TO 2781 01:43:12,523 --> 01:43:13,524 INTELLECTUAL PROPERTY, MAKING 2782 01:43:13,524 --> 01:43:15,292 SURE THE COMPANY HAS ACCESS TO 2783 01:43:15,292 --> 01:43:17,962 THE DATA AND THE LIABILITY AND 2784 01:43:17,962 --> 01:43:18,963 RESPONSIBILITY OBLIGATIONS FIT 2785 01:43:18,963 --> 01:43:20,965 THE TYPE OF STUDY WE ARE 2786 01:43:20,965 --> 01:43:21,732 NEGOTIATING FOR. 2787 01:43:21,732 --> 01:43:27,438 SO YOU CAN GO TO THE NEXT SLIDE. 2788 01:43:27,438 --> 01:43:29,607 SO I COULD PROBABLY TALK FOR 20 2789 01:43:29,607 --> 01:43:31,108 MINUTES ABOUT INTELLECTUAL 2790 01:43:31,108 --> 01:43:32,743 PROPERTY PROVISIONS IN 2791 01:43:32,743 --> 01:43:34,645 CONTRACTS, BUT I THINK THE BIG 2792 01:43:34,645 --> 01:43:36,347 TAKEAWAY IS THAT THE COMPANY 2793 01:43:36,347 --> 01:43:38,515 DOESN'T FEEL LIKE THEY ARE BEING 2794 01:43:38,515 --> 01:43:41,352 BLOCKED FROM POTENTIAL 2795 01:43:41,352 --> 01:43:42,486 INVENTIONS THAT ARE AN 2796 01:43:42,486 --> 01:43:43,921 IMPROVEMENT OR THE USE OF T 2797 01:43:43,921 --> 01:43:45,656 Y E INVESTED SO MUCH 2798 01:43:45,656 --> 01:43:49,593 TIME AND MONEY INTO. 2799 01:43:49,593 --> 01:43:52,196 AND I THINK THE MOST COMMON WAY 2800 01:43:52,196 --> 01:43:55,099 TO STRUCTURE THIS CONTRACTUALLY 2801 01:43:55,099 --> 01:43:59,303 IS TO STUDY DRUG IP SEPARATED 2802 01:43:59,303 --> 01:44:00,738 OUT FROM OTHER IP. 2803 01:44:00,738 --> 01:44:02,339 AS LONG AS THE COMPANY FEELS 2804 01:44:02,339 --> 01:44:03,907 LIKE THEY HAVE OWNERSHIP RIGHTS 2805 01:44:03,907 --> 01:44:07,211 OR EXCLUSIVITY TO THOSE STUDY 2806 01:44:07,211 --> 01:44:09,780 DRUG RELATED INVENTIONS THERE IS 2807 01:44:09,780 --> 01:44:12,816 GENERALLY A PATH FORWARD. 2808 01:44:12,816 --> 01:44:14,018 AND THIS IS ANOTHER REASON TO 2809 01:44:14,018 --> 01:44:15,219 PICK UP THE PHONE AND MAKE SURE 2810 01:44:15,219 --> 01:44:17,655 WE ARE ALIGNED ON THE INTERESTS 2811 01:44:17,655 --> 01:44:18,656 OF BOTH PARTIES. 2812 01:44:18,656 --> 01:44:21,125 THE BIG, ONE OF THE BIG 2813 01:44:21,125 --> 01:44:22,860 DIFFERENCES BETWEEN 2814 01:44:22,860 --> 01:44:24,161 INVESTIGATOR-INITIATED TRIAL 2815 01:44:24,161 --> 01:44:26,430 CONTRACTS AND COMPANY-SPONSORED 2816 01:44:26,430 --> 01:44:28,499 TRIAL CONTRACTS IS THE LIABILITY 2817 01:44:28,499 --> 01:44:29,133 PROVISION, RIGHT? 2818 01:44:29,133 --> 01:44:32,903 I HAVE SEEN A LOT OF CONTRACT 2819 01:44:32,903 --> 01:44:36,840 NEGOTIATIONS WHERE THE SPONSOR 2820 01:44:36,840 --> 01:44:37,541 INSTITUTION IS ASKING FOR 2821 01:44:37,541 --> 01:44:41,845 LIABILITY TERMS THAT ARE SIMILAR 2822 01:44:41,845 --> 01:44:43,681 COMPANY-SPONSORED CONTRACTSRE 2823 01:44:43,681 --> 01:44:44,982 MEANING THEY ARE ASKING THE 2824 01:44:44,982 --> 01:44:46,050 COMPANY WHO ME SUPPORTING 2825 01:44:46,050 --> 01:44:48,052 THE STUDY TO EITHER PROVIDE 2826 01:44:48,052 --> 01:44:52,423 INSURANCE OR BE LIABLE FOR 2827 01:44:52,423 --> 01:44:53,390 SUBJECT INJURY. 2828 01:44:53,390 --> 01:44:56,126 FOR AN INVESTIGATOR-INITIATED 2829 01:44:56,126 --> 01:44:57,795 TRIAL WHERE THE COMPANY IS NOT 2830 01:44:57,795 --> 01:44:59,363 CONTROLLING THE DESIG OR 2831 01:44:59,363 --> 01:45:02,766 CONDUCT, TYPICALLY THE MOST IN 2832 01:45:02,766 --> 01:45:05,069 TERMSINDEMNIFICATION 2833 01:45:05,069 --> 01:45:06,770 RELIABILITY WE CAN PROVIDE IS 2834 01:45:06,770 --> 01:45:08,972 ANY INJURY CAUSED BY MAYBE 2835 01:45:08,972 --> 01:45:10,541 MANUFACTURING DEFECTS OR 2836 01:45:10,541 --> 01:45:12,876 LABELING DEFECTS TO THE DRUG. 2837 01:45:12,876 --> 01:45:17,748 THE ACCESS TO THE DATA IS REALLY 2838 01:45:17,748 --> 01:45:17,915 KEY. 2839 01:45:17,915 --> 01:45:22,720 I'VE SEEN COMPANIES ASK FOR 2840 01:45:22,720 --> 01:45:23,787 REGULAR REPORTS, QUARTERLY 2841 01:45:23,787 --> 01:45:30,761 REPORTS ON THE STATUS OF THE 2842 01:45:30,761 --> 01:45:33,097 TRIAL AND THE COMPANY AND THE 2843 01:45:33,097 --> 01:45:34,698 SPONSOR W DETERMINE TOGETHER 2844 01:45:34,698 --> 01:45:36,300 WHAT THE SAFETY REPORTING LOOKS 2845 01:45:36,300 --> 01:45:36,500 LIKE. 2846 01:45:36,500 --> 01:45:37,234 IT I A BIG PART OF THE 2847 01:45:37,234 --> 01:45:37,701 CONTRACT. 2848 01:45:37,701 --> 01:45:42,506 ACCESS TO DATA IS BIG. 2849 01:45:42,506 --> 01:45:44,875 THERE ARE SOME COUNTRIES WHERE 2850 01:45:44,875 --> 01:45:48,312 THE COMPANY WOULD HAVE TO PAY AN 2851 01:45:48,312 --> 01:45:51,548 ADDITIONAL FEE OR LICENSE THE 2852 01:45:51,548 --> 01:45:51,749 DATA. 2853 01:45:51,749 --> 01:45:53,417 AND THOSE COUNTRIES ARE 2854 01:45:53,417 --> 01:45:56,754 TYPICALLY AVOIDED IN 2855 01:45:56,754 --> 01:45:57,888 INVESTIGATOR-INITIATED TRIAL 2856 01:45:57,888 --> 01:46:00,924 SUPPORT ON THE COMPANY SIDE. 2857 01:46:00,924 --> 01:46:03,026 TERMINATION OBLIGATIONS ARE 2858 01:46:03,026 --> 01:46:06,864 ANOTHER CONSIDERATION FOR DRUG 2859 01:46:06,864 --> 01:46:07,231 COMPANIES. 2860 01:46:07,231 --> 01:46:08,565 BOTH, I THINK BOTH THE SPONSOR 2861 01:46:08,565 --> 01:46:10,234 AND THE COMPANIES ARE ALIGNED 2862 01:46:10,234 --> 01:46:12,536 THAT THE TRIAL MIGHT BE 2863 01:46:12,536 --> 01:46:14,371 TERMINATED FOR SAFETY REASONS OR 2864 01:46:14,371 --> 01:46:16,440 POTENTIALLY FOR RFUTIR BUT 2865 01:46:16,440 --> 01:46:17,508 SOMETIMES SUPPLY CONSIDERATIONS 2866 01:46:17,508 --> 01:46:20,244 COME INTO PLAY. 2867 01:46:20,244 --> 01:46:21,645 IF THE COMPANY NO LONGER 2868 01:46:21,645 --> 01:46:23,413 MANUFACTURING A DRUG, THEY ARE 2869 01:46:23,413 --> 01:46:24,748 PROBABLY NOT GOING TO 2870 01:46:24,748 --> 01:46:26,617 MANUFACTURE THE DRUG 2871 01:46:26,617 --> 01:46:29,720 SPECIFICALLY FOR THE 2872 01:46:29,720 --> 01:46:30,854 INVESTIGATOR-INITIATED TRIAL. 2873 01:46:30,854 --> 01:46:32,923 I THINK, YOU KNOW, SOMETIMES 2874 01:46:32,923 --> 01:46:34,258 ENROLLMENT JUST DOESN'T HAPPEN 2875 01:46:34,258 --> 01:46:35,626 THE WAY THAT THE SPONSOR 2876 01:46:35,626 --> 01:46:38,428 INTENDED. 2877 01:46:38,428 --> 01:46:39,530 SO, YOU KNOW, WITH FUNDING TIED 2878 01:46:39,530 --> 01:46:42,800 TO ENROLLMENT, SOMETIMES A 2879 01:46:42,800 --> 01:46:45,402 COMPANY MIGHT NEED TO EXERCISE 2880 01:46:45,402 --> 01:46:46,603 TERMINATION RIGHTS BECAUSE OF 2881 01:46:46,603 --> 01:46:57,080 TIME LIMITS AND ENROLLMENT. 2882 01:47:00,384 --> 01:47:03,987 I HAVE BEEN INVOLVED WITH TRIALS 2883 01:47:03,987 --> 01:47:05,823 THAT INVOLVED MORE THAN ONE DRUG 2884 01:47:05,823 --> 01:47:06,156 COMPANY. 2885 01:47:06,156 --> 01:47:08,358 IT MIGHT BE A STUDY DESIGNED 2886 01:47:08,358 --> 01:47:09,927 WITH MULTIPLE ARMS. 2887 01:47:09,927 --> 01:47:11,895 FOR CONTRACTING, I WOULD 2888 01:47:11,895 --> 01:47:13,997 RECOMMEND THAT BOTH OR MORE THAN 2889 01:47:13,997 --> 01:47:16,166 TWO DRUG COMPANIES ALIGN FIRST 2890 01:47:16,166 --> 01:47:22,439 NEGOTIATING WITH THE 2891 01:47:22,439 --> 01:47:24,141 SPONSOR INSTITUTION. 2892 01:47:24,141 --> 01:47:25,409 TYPICALLY A SMALLER COMPANY 2893 01:47:25,409 --> 01:47:26,410 WOULD BE HAPPY TO USE THE 2894 01:47:26,410 --> 01:47:28,412 CONTRACT FOR A LARGER COMPANY SO 2895 01:47:28,412 --> 01:47:31,682 LET THEM SORT OFOR ALIGN FIRST. 2896 01:47:31,682 --> 01:47:34,284 IF EVERYONE IS CONTRACTING 2897 01:47:34,284 --> 01:47:35,686 SEPARATELY, THE MAIN THING I 2898 01:47:35,686 --> 01:47:38,522 LOOK FOR ISHAT THE 2899 01:47:38,522 --> 01:47:39,356 INSTITUTION, THE SPONSOR 2900 01:47:39,356 --> 01:47:40,791 INSTITUTION IS GOING TO BE ABLE 2901 01:47:40,791 --> 01:47:43,493 TO REPRESENT THE IP TERMS, 2902 01:47:43,493 --> 01:47:44,828 INTELLECTUAL PROPERTY TERMS FOR 2903 01:47:44,828 --> 01:47:49,299 ALL PARTIES ARE ALIGNED AND 2904 01:47:49,299 --> 01:47:50,601 CONSISTENT AND NEITHER COMPANY 2905 01:47:50,601 --> 01:47:52,035 IS GOING TO HAVE MORE FAVORABLE 2906 01:47:52,035 --> 01:47:53,971 OR QUICKER TRANSMISSION OF THE 2907 01:47:53,971 --> 01:47:54,371 DATA. 2908 01:47:54,371 --> 01:47:55,772 SO WE WOULDN'T WANT ANOTHER 2909 01:47:55,772 --> 01:47:56,640 COMPANY KNOWING ABOUT THE DATA 2910 01:47:56,640 --> 01:48:03,747 BEFORE WE DO. 2911 01:48:03,747 --> 01:48:06,383 SO FOR INVESTIGATOR-INITIATED 2912 01:48:06,383 --> 01:48:08,285 TRIALS THAT THE COMPANY IS 2913 01:48:08,285 --> 01:48:11,054 SUPPORTING WITH FUNDING, WE 2914 01:48:11,054 --> 01:48:12,489 TYPICALLY SEE THE BUDGET 2915 01:48:12,489 --> 01:48:13,891 STRUCTURE, THE PAYMENT TERMS 2916 01:48:13,891 --> 01:48:15,726 THAT ARE MILESTONE BASED AND 2917 01:48:15,726 --> 01:48:17,261 TYPICALLY FOR ENROLLMENT. 2918 01:48:17,261 --> 01:48:23,066 SO WE MIGHT SEE THE FIRST 2919 01:48:23,066 --> 01:48:25,669 PAYMENT BE -- DUE AFTER CONTRACT 2920 01:48:25,669 --> 01:48:28,839 EXECUTION AND SEVERAL ENROLLMENT 2921 01:48:28,839 --> 01:48:29,773 BASED MILESTONES. 2922 01:48:29,773 --> 01:48:31,108 TYPICALLY THE LAST MILESTONE 2923 01:48:31,108 --> 01:48:33,477 PAYMENT WILL BE PAID OUT AFTER 2924 01:48:33,477 --> 01:48:36,546 COMPLETION OF THE FINAL REPORT 2925 01:48:36,546 --> 01:48:40,517 OR A MANUSCRIPT SUBMISSION. 2926 01:48:40,517 --> 01:48:42,853 ONE THING I LIKE TO HIGHLIGHT IS 2927 01:48:42,853 --> 01:48:44,922 WHERE YOU ARE TALKING ABOUT AN 2928 01:48:44,922 --> 01:48:45,989 INVESTIGATOR INITIATED TRIAL AND 2929 01:48:45,989 --> 01:48:50,093 THE COMPANY IS ARM'S LENGTH, I 2930 01:48:50,093 --> 01:48:52,529 RECOMMEND A LINE ITEM FOR 2931 01:48:52,529 --> 01:48:53,563 PUBLICATION SUPPORT. 2932 01:48:53,563 --> 01:48:56,033 I HAVE SEEN REQUESTS FOR 2933 01:48:56,033 --> 01:48:57,367 PUBLICATION SUPPORT COME AFTER 2934 01:48:57,367 --> 01:48:59,069 THE DATA HAS BEEN GENERATED AND 2935 01:48:59,069 --> 01:49:01,738 I THINK THAT THE COMPANY IS MUCH 2936 01:49:01,738 --> 01:49:05,676 LESS LIKELY TO SUPPORT PAY FOR 2937 01:49:05,676 --> 01:49:07,878 PUBLICATION AFTER THE FACT SO IT 2938 01:49:07,878 --> 01:49:09,579 DOESN'T LOOK LIKE MAYBE THEY ARE 2939 01:49:09,579 --> 01:49:14,818 PAYING JUST FOR GOOD DATA. 2940 01:49:14,818 --> 01:49:15,419 NEXT SLIDE. 2941 01:49:15,419 --> 01:49:18,655 SO IT IS PRETTY COMMON FOR A 2942 01:49:18,655 --> 01:49:20,657 SPONSOR INVESTIGATOR TO BRING ON 2943 01:49:20,657 --> 01:49:23,560 SUB SITES, ESPECIALLY IN A RARE 2944 01:49:23,560 --> 01:49:25,595 DISEASE SPACE WHERE THERE ARE 2945 01:49:25,595 --> 01:49:29,099 ENROLLMENT LIMITATIONS AT A 2946 01:49:29,099 --> 01:49:31,001 PARTICULAR SITE. 2947 01:49:31,001 --> 01:49:32,069 TYPICALLY THE DRUG COMPANY 2948 01:49:32,069 --> 01:49:34,004 DOESN'T GET INVOLVED AT ALL WITH 2949 01:49:34,004 --> 01:49:35,672 CONTRACTING FOR SUB SITES. 2950 01:49:35,672 --> 01:49:37,174 WE LOOK FOR REPRESENTATION THAT 2951 01:49:37,174 --> 01:49:38,842 THE SPONSOR INSTITUTION IS GOING 2952 01:49:38,842 --> 01:49:40,711 TO ENSURE THAT ANY SUB SITE 2953 01:49:40,711 --> 01:49:44,781 COMPLIES WITH THE SAME TERMS AS 2954 01:49:44,781 --> 01:49:45,349 OUR CONTRACT. 2955 01:49:45,349 --> 01:49:46,917 SO THAT IS ALL SORT OF DONE 2956 01:49:46,917 --> 01:49:49,019 BEHIND THE SCENES AND IF THERE 2957 01:49:49,019 --> 01:49:52,456 ARE ANY ISSUES WE GO DIRECTLY TO 2958 01:49:52,456 --> 01:49:53,890 THE SPONSOR. 2959 01:49:53,890 --> 01:49:57,627 WE, AS A SPONSOR -- SORRY, WE AS 2960 01:49:57,627 --> 01:49:59,429 THE DRUG MANUFACTURE MIGHT AGREE 2961 01:49:59,429 --> 01:50:00,931 TO SHIP THE DRUG TO SUB SITES 2962 01:50:00,931 --> 01:50:03,133 AND THAT CAN BE WORKED OUT IN 2963 01:50:03,133 --> 01:50:03,934 THE CONTRACT. 2964 01:50:03,934 --> 01:50:05,769 USUALLY THERE IS A DRUG SUPPLY 2965 01:50:05,769 --> 01:50:08,638 FORM WE ATTACH AS AN APPENDIX. 2966 01:50:08,638 --> 01:50:12,376 WE ARE ALIGNED ON WHAT THAT FORM 2967 01:50:12,376 --> 01:50:14,411 LOOKS LIKE AND HOW WE GET DRUG 2968 01:50:14,411 --> 01:50:15,979 TO THE VARIOUS SUB SITES. 2969 01:50:15,979 --> 01:50:17,481 I MENTIONED EARLIER THERE ARE 2970 01:50:17,481 --> 01:50:19,349 SOME COUNTRIES THAT MIGHT NOT 2971 01:50:19,349 --> 01:50:21,952 WORK TO HAVE SUB SITES IN. 2972 01:50:21,952 --> 01:50:24,521 SEVERAL EUROPEAN COUNTRIES 2973 01:50:24,521 --> 01:50:27,057 WOULDN'T BE ABLE TO AGREE TO THE 2974 01:50:27,057 --> 01:50:30,560 TERMS THAT A COMPANY REQUIRES IN 2975 01:50:30,560 --> 01:50:34,031 TERMS OF LICENSING OR ASSIGNMENT 2976 01:50:34,031 --> 01:50:34,664 OF INTELLECTUAL PROPERTY AND 2977 01:50:34,664 --> 01:50:36,166 ALSO THE ACCESS TO THE DATA. 2978 01:50:36,166 --> 01:50:38,001 SO WE MIGHT BE ABLE TO GET A 2979 01:50:38,001 --> 01:50:39,703 HIGH LEVEL SUMMARY, BUT IN TERMS 2980 01:50:39,703 --> 01:50:42,239 OF ACCESS DATA WE WOULD HAVE 2981 01:50:42,239 --> 01:50:45,275 PAY FOR THAT, WHICH TYPICALLY 2982 01:50:45,275 --> 01:50:48,812 CANNOTE NEGOTIATED UPFRONT. 2983 01:50:48,812 --> 01:50:51,715 BECAUSE THE VALUE OF THE DATA OR 2984 01:50:51,715 --> 01:50:53,350 THE INVENTION CAN'T BE 2985 01:50:53,350 --> 01:50:53,683 PREDETERMINED. 2986 01:50:53,683 --> 01:50:55,452 SO COMPANIES FOR THE MOST PART 2987 01:50:55,452 --> 01:50:58,221 STAY AWAY FROM SUPPORTING 2988 01:50:58,221 --> 01:50:59,589 INVESTIGATOR-INITIATED STUDIES 2989 01:50:59,589 --> 01:51:01,858 IN THOSE COUNTRIES WHERE THOSE 2990 01:51:01,858 --> 01:51:02,292 RESTRICTIONS EXIST. 2991 01:51:02,292 --> 01:51:04,861 SO I THINK IT IS IMPORTANT TO 2992 01:51:04,861 --> 01:51:05,962 UNDERSTAND WHERE THERS POTENTIA 2993 01:51:05,962 --> 01:51:09,800 SUB SITES ALSO MIGHT BE FROM THE 2994 01:51:09,800 --> 01:51:11,535 SPONSOR AT THE GET-GO TO MAKE 2995 01:51:11,535 --> 01:51:12,969 SURE WE ARE NOT GOING TO HAVE 2996 01:51:12,969 --> 01:51:14,971 ANY ISSUES WITH SUB SITES COME 2997 01:51:14,971 --> 01:51:21,111 PLYI II IING -- COMPLYING WITH 2998 01:51:21,111 --> 01:51:22,546 TERMS OF THE CONTRACT. 2999 01:51:22,546 --> 01:51:25,515 I THINK THAT IS THE LAST SLIDE. 3000 01:51:25,515 --> 01:51:26,049 >> THANK YOU, KAREN. 3001 01:51:26,049 --> 01:51:27,417 >> YEAH. 3002 01:51:27,417 --> 01:51:30,187 >> SO IF WE CAN GO AHEAD AND 3003 01:51:30,187 --> 01:51:33,290 HAVE ALL OF OUR SPEAKERS FROM 3004 01:51:33,290 --> 01:51:34,791 SESSION 4 TURN ON YOUR CAMERAS 3005 01:51:34,791 --> 01:51:37,027 SO YOU CAN BE PRESENT FOR 3006 01:51:37,027 --> 01:51:37,327 QUESTIONS. 3007 01:51:37,327 --> 01:51:39,296 THEY WILL HIGHLIGHT US SO WE ARE 3008 01:51:39,296 --> 01:51:41,631 ONLY THE ONES SEEN TALKING. 3009 01:51:41,631 --> 01:51:43,400 AND REMINDER TO EVERYBODY TO PUT 3010 01:51:43,400 --> 01:51:44,968 QUESTIONS IN THE Q&ABOX. 3011 01:51:44,968 --> 01:51:46,837 WE DEFINITELY HAVE SOME WE CAN 3012 01:51:46,837 --> 01:51:48,738 GOAD AND GET STARTED WITH. 3013 01:51:48,738 --> 01:51:50,173 FIRST OF ALL, THANK YOU. 3014 01:51:50,173 --> 01:51:51,808 IT IS A NICE PICTURE WE HAVE 3015 01:51:51,808 --> 01:51:53,276 PAINTED OVER THE MORNING FROM 3016 01:51:53,276 --> 01:51:54,644 SPECIAL CONSIDERATIONS TO 3017 01:51:54,644 --> 01:51:57,714 ACTUALLY THINKING ABOUT HOW TO 3018 01:51:57,714 --> 01:51:59,382 DESIGN A TRIAL TO, HEY, WE DID 3019 01:51:59,382 --> 01:52:01,184 THIS TRIAL, THIS IS WHAT WE 3020 01:52:01,184 --> 01:52:02,486 LEARNED AND THE IMPORTANT WORK 3021 01:52:02,486 --> 01:52:03,954 THAT YOU ARE DOING ON THE BACK 3022 01:52:03,954 --> 01:52:06,890 END, KAREN, OF MAKING SURE THAT 3023 01:52:06,890 --> 01:52:08,558 ALL THE CONTRACTS LANGUAGE COMES 3024 01:52:08,558 --> 01:52:09,326 TOGETHER SO IT IS ACTUALLY 3025 01:52:09,326 --> 01:52:11,428 SUCCESSFUL. 3026 01:52:11,428 --> 01:52:13,196 SO I WILL GO AHEAD AND START 3027 01:52:13,196 --> 01:52:14,664 WITH SOME OF THE QUESTIONS IN 3028 01:52:14,664 --> 01:52:15,932 THE CHAT AND CERTAINLY MORE CAN 3029 01:52:15,932 --> 01:52:19,703 COME IN. 3030 01:52:19,703 --> 01:52:22,672 JEFF MOFFIT BROUGHT UP THE 3031 01:52:22,672 --> 01:52:26,176 UPCOMING APPROVAL OF NURANDOMA 3032 01:52:26,176 --> 01:52:28,545 LARKS AST, WHICH I'M PROBABLY 3033 01:52:28,545 --> 01:52:31,248 NOT SAYING RIGHT, A NEW DRUG 3034 01:52:31,248 --> 01:52:35,285 COMING TO THE MARKET FOR 3035 01:52:35,285 --> 01:52:37,120 IDIOPATHIC FIBROSIS. 3036 01:52:37,120 --> 01:52:38,488 DOES THAT CHANGE HOW WE ARE 3037 01:52:38,488 --> 01:52:40,090 THINKING OF NINTEDANIB IN 3038 01:52:40,090 --> 01:52:41,424 CHILDREN AND HOW DO WE THINK 3039 01:52:41,424 --> 01:52:44,294 ABOUT THIS CONCEPT MORE 3040 01:52:44,294 --> 01:52:44,661 GENERALLY? 3041 01:52:44,661 --> 01:52:46,563 NEW APPROVAL THAT MAY REBALANCE 3042 01:52:46,563 --> 01:52:48,498 THAT CLINICAL RISK BENEFIT. 3043 01:52:48,498 --> 01:52:50,333 >> I ASSUME THAT IS MY QUESTION? 3044 01:52:50,333 --> 01:52:50,567 >> YEAH. 3045 01:52:50,567 --> 01:52:52,202 I THINK SO. 3046 01:52:52,202 --> 01:52:56,173 SO THERE IS A LOT OF PIPELINE 3047 01:52:56,173 --> 01:52:56,907 POTENTIAL DRUG. 3048 01:52:56,907 --> 01:53:02,145 NOT EVERYTHING MAKES IT THROUGH 3049 01:53:02,145 --> 01:53:05,115 PHASE 3. 3050 01:53:05,115 --> 01:53:12,322 THAT IS AH PLAN TO APPROACH OUR 3051 01:53:12,322 --> 01:53:12,923 COMMUNITY AGAIN. 3052 01:53:12,923 --> 01:53:15,225 ONE OF THE THINGS ABOUT THE EMA 3053 01:53:15,225 --> 01:53:16,927 APPROVAL IS WE HAVE AT LEAST A 3054 01:53:16,927 --> 01:53:18,662 STARTING PLACE FOR A DESIGN TO 3055 01:53:18,662 --> 01:53:21,064 THINK ABOUT ONE THAT WAS 3056 01:53:21,064 --> 01:53:21,364 SUCCESSFUL. 3057 01:53:21,364 --> 01:53:23,800 THERE COULD BE MODIFICATIONS TO 3058 01:53:23,800 --> 01:53:24,000 THAT. 3059 01:53:24,000 --> 01:53:26,937 BUT WE HAVE A STARTING PLACE TO 3060 01:53:26,937 --> 01:53:29,172 BE ABLE TO THINK ABOUT STUDYING 3061 01:53:29,172 --> 01:53:30,540 THIS OTHER MEDICATION. 3062 01:53:30,540 --> 01:53:32,209 THERE ARE MANY THINGS TO THINK 3063 01:53:32,209 --> 01:53:35,045 ABOUT AS NEW DRUGS COME FORWARD 3064 01:53:35,045 --> 01:53:35,979 IN THE ADULT PIPELINE. 3065 01:53:35,979 --> 01:53:37,714 WE STILL HAVE A LIMITED NUMBER 3066 01:53:37,714 --> 01:53:39,316 OF PATIENTS. 3067 01:53:39,316 --> 01:53:41,952 SO HOW DO YOU CHOOSE WHICH 3068 01:53:41,952 --> 01:53:43,453 DRUGS? 3069 01:53:43,453 --> 01:53:45,255 HOW DO YOU INCLUDE -- DO THEY 3070 01:53:45,255 --> 01:53:48,625 GET TO BE ON NINTEDANIB? 3071 01:53:48,625 --> 01:53:50,560 DO YOU CHOOSE MANY IN THE 3072 01:53:50,560 --> 01:53:52,862 CLINICAL REALM? 3073 01:53:52,862 --> 01:53:58,435 DO YOU CHOOSE NINTEDANIB -- HAS 3074 01:53:58,435 --> 01:53:59,502 AN INDICATION. 3075 01:53:59,502 --> 01:54:03,073 ARE YOU GOING TO DO OFF LABEL? 3076 01:54:03,073 --> 01:54:04,608 OUR COMMUNITY WILL BE APPROACHED 3077 01:54:04,608 --> 01:54:07,177 BECAUSE THERE WILL BE PEPS 3078 01:54:07,177 --> 01:54:08,011 INVESTAL PLANS. 3079 01:54:08,011 --> 01:54:10,413 WE HAVE TO THINK OF THE SIZE OF 3080 01:54:10,413 --> 01:54:13,149 OUR COMMUNITY, HOW WE DESIGN 3081 01:54:13,149 --> 01:54:14,284 THESE TRIALS. 3082 01:54:14,284 --> 01:54:16,119 DID WE LEARN THINGS THAT MAKE 3083 01:54:16,119 --> 01:54:17,287 THE NEXT TRIAL EASIER? 3084 01:54:17,287 --> 01:54:19,422 I THINK WE DID, I THINK WE HAVE 3085 01:54:19,422 --> 01:54:21,057 AND ARE WORKING QUICKLY TO 3086 01:54:21,057 --> 01:54:24,060 ESTABLISH SOME OF THOSE 3087 01:54:24,060 --> 01:54:24,461 ASSUMPTIONS. 3088 01:54:24,461 --> 01:54:26,329 WE HAVE REGULATORY WORK TO DO 3089 01:54:26,329 --> 01:54:28,298 WITH THE FDA. 3090 01:54:28,298 --> 01:54:29,232 NINTEDANIB'S PATHWAY FORWARD, 3091 01:54:29,232 --> 01:54:31,935 THAT WAS A FINAL LETTER FROM THE 3092 01:54:31,935 --> 01:54:32,102 FDA. 3093 01:54:32,102 --> 01:54:35,905 SO NEW DRUG MEDICATIONS STUDIES 3094 01:54:35,905 --> 01:54:38,241 WILL HOPEFULLY OPEN PATHWAYS TO 3095 01:54:38,241 --> 01:54:39,876 THE FDA. 3096 01:54:39,876 --> 01:54:42,746 AND THAT PROCESS, THAT WE CAN 3097 01:54:42,746 --> 01:54:48,285 HAVE BROADER CONVERSATIONS. 3098 01:54:48,285 --> 01:54:50,787 >> AND I WANTED TO FOLLOW UP TO 3099 01:54:50,787 --> 01:54:53,189 THE EXTENT YOU CAN SHARE YOUR 3100 01:54:53,189 --> 01:54:56,159 INSIGHTS INTO WHY THE APPROVAL 3101 01:54:56,159 --> 01:54:59,429 FROM THE EMA VERSUS THE FDA AND 3102 01:54:59,429 --> 01:55:05,335 I KNOW YOU CAN'T SHARE SOME 3103 01:55:05,335 --> 01:55:06,069 SPECIFIC 3104 01:55:06,069 --> 01:55:06,803 SPECIFIC 3105 01:55:06,803 --> 01:55:07,337 SPECI 3106 01:55:07,337 --> 01:55:07,904 SPECIFICS, HOW MIGHT YOUR 3107 01:55:07,904 --> 01:55:08,938 APPROACH BE DIFFERENT IN TRIAL 3108 01:55:08,938 --> 01:55:09,406 DEVELOPMENT? 3109 01:55:09,406 --> 01:55:11,274 >> I'M ALWAYS NERVOUS WHAT I CAN 3110 01:55:11,274 --> 01:55:12,642 SAY AND CAN'T SAY. 3111 01:55:12,642 --> 01:55:14,044 WE HAD FOLLOW-UP CONVERSATIONS 3112 01:55:14,044 --> 01:55:16,379 WITH MEMBERS OF THE FDA. 3113 01:55:16,379 --> 01:55:18,548 WE HAVE WORK TO DO IN THINKING 3114 01:55:18,548 --> 01:55:22,919 ABOUT PEDIATRICS AND ADULTS. 3115 01:55:22,919 --> 01:55:25,221 BAYESIAN ANALYSIS, WE BORROWED 3116 01:55:25,221 --> 01:55:27,190 THE POPULATION FROM THE ADULT 3117 01:55:27,190 --> 01:55:28,158 STUDIES. 3118 01:55:28,158 --> 01:55:30,093 WHAT IS THE BEST GROUP OF ADULT 3119 01:55:30,093 --> 01:55:32,162 PATIENTS TO BORROW FROM? 3120 01:55:32,162 --> 01:55:34,297 PROBABLY NOT IPF, BUT THERE ARE 3121 01:55:34,297 --> 01:55:34,931 OTHERS. 3122 01:55:34,931 --> 01:55:37,901 SO HOW DO WE REFINE OUR 3123 01:55:37,901 --> 01:55:39,903 CONVERSATIONS ABOUT MAKING 3124 01:55:39,903 --> 01:55:40,970 COMPARISONS TO THE ADULT 3125 01:55:40,970 --> 01:55:41,338 POPULATION? 3126 01:55:41,338 --> 01:55:44,407 HOW DO WE DO RESEARCH TO LOOK AT 3127 01:55:44,407 --> 01:55:47,077 HOW DOES PEDIATRIC FIBROSIS 3128 01:55:47,077 --> 01:55:51,448 COMPARE TO ADULT FIBROSIS. 3129 01:55:51,448 --> 01:55:54,050 NOW IT IS A CRITICAL QUESTION TO 3130 01:55:54,050 --> 01:55:55,819 BRING THESE TRIALS FORWARD. 3131 01:55:55,819 --> 01:55:58,254 I BELIEVE MYSELF THE MECHANISM 3132 01:55:58,254 --> 01:56:00,924 OF DISEASE WITH FIBROSIS, THOSE 3133 01:56:00,924 --> 01:56:02,392 PATHWAYS ARE STILL ACTIVE IN 3134 01:56:02,392 --> 01:56:04,894 KIDS AND ADULTS, BUT THE 3135 01:56:04,894 --> 01:56:06,162 BACKGROUND OF DEVELOPMENT IS 3136 01:56:06,162 --> 01:56:06,463 IMPORTANT. 3137 01:56:06,463 --> 01:56:07,263 THINKING DEVELOPMENTALLY ABOUT 3138 01:56:07,263 --> 01:56:07,464 THIS. 3139 01:56:07,464 --> 01:56:09,332 THERE ARE A LOT OF QUESTIONS 3140 01:56:09,332 --> 01:56:11,534 THAT CAN BE ASKED IN BASIC 3141 01:56:11,534 --> 01:56:13,570 SCIENCE AND TRANSLATIONAL ARENA 3142 01:56:13,570 --> 01:56:14,871 THAT ARE NOW CRITICAL BECAUSE 3143 01:56:14,871 --> 01:56:16,106 THEY ARE GOING TO BE IMPORTANT 3144 01:56:16,106 --> 01:56:19,142 FOR US GETTING TREATMENT TO 3145 01:56:19,142 --> 01:56:20,577 KIDS. 3146 01:56:20,577 --> 01:56:24,180 IMAGING PATHOLOGIC CORRELATION 3147 01:56:24,180 --> 01:56:27,484 TO VALIDATE CT CRITERIA ARE 3148 01:56:27,484 --> 01:56:28,885 STUDIES UNDER WAY. 3149 01:56:28,885 --> 01:56:30,286 I THINK THE FDA WANTS TO SEE 3150 01:56:30,286 --> 01:56:35,325 SOME OF THAT TO UNDERSTAND THOSE 3151 01:56:35,325 --> 01:56:36,126 DIFFERENCES. 3152 01:56:36,126 --> 01:56:37,594 AND WE -- THE PATIENT COMMUNITY 3153 01:56:37,594 --> 01:56:39,596 MAYBE NEEDS TO BE MORE ACTIVE 3154 01:56:39,596 --> 01:56:41,164 ABOUT WE NEED THESE DRUGS. 3155 01:56:41,164 --> 01:56:42,999 HOW SAFE DO YOU WANT TO BE 3156 01:56:42,999 --> 01:56:45,368 COMPARED TO GETTING DRUGS TO 3157 01:56:45,368 --> 01:56:48,538 KIDS OR NOT GETTING DRUGS TO 3158 01:56:48,538 --> 01:56:50,073 KIDS THAT ARE DYING FROM THIS? 3159 01:56:50,073 --> 01:56:52,909 WHAT IS THE RISK BENEFIT RATIO 3160 01:56:52,909 --> 01:56:53,910 WE CAN TALK ABOUT? 3161 01:56:53,910 --> 01:56:57,113 THERE IS ADVOCACY WORK. 3162 01:56:57,113 --> 01:56:57,781 SCIENTIFIC INVESTIGATIONS WORK 3163 01:56:57,781 --> 01:56:59,449 THAT WILL NEED TO COME FORWARD. 3164 01:56:59,449 --> 01:57:01,484 AND NOW THERE WILL BE DIFFERENT 3165 01:57:01,484 --> 01:57:03,119 CONVERSATIONS NOW THE DESIGN HAS 3166 01:57:03,119 --> 01:57:04,988 BEEN AT LEAST VALIDATED IN 3167 01:57:04,988 --> 01:57:07,524 EUROPE. 3168 01:57:07,524 --> 01:57:08,324 BUT I'M OPEN. 3169 01:57:08,324 --> 01:57:11,194 I HAVE EXPERTS ON THE LINE THAT 3170 01:57:11,194 --> 01:57:12,462 COULD TAKE US FURTHER WITH 3171 01:57:12,462 --> 01:57:14,564 INSIGHTS SO I APPRECIATE THAT. 3172 01:57:14,564 --> 01:57:16,599 >> DORIS, FROM YOUR PERSPECTIVE 3173 01:57:16,599 --> 01:57:18,234 AS A CONSULTANT IN THIS SPACE, 3174 01:57:18,234 --> 01:57:20,003 WHEN YOU ARE WORKING WITH SOME 3175 01:57:20,003 --> 01:57:22,639 OF US MAYBE IN THE ROOM OR 3176 01:57:22,639 --> 01:57:24,674 TRYING TO THINK ABOUT HOW IF WE 3177 01:57:24,674 --> 01:57:26,075 NEED TO LAY THE GROUNDWORK FOR 3178 01:57:26,075 --> 01:57:29,979 THE END POINTS WE ARE USING FOR 3179 01:57:29,979 --> 01:57:31,247 STUDIES FOR RARE LUNG DISEASE IN 3180 01:57:31,247 --> 01:57:34,317 KIDS WHAT FACTORS DO WE NEED TO 3181 01:57:34,317 --> 01:57:35,452 CONSIDER WHEN WE THINK ABOUT 3182 01:57:35,452 --> 01:57:36,753 EVENTUALLY GETTING FDA APPROVAL? 3183 01:57:36,753 --> 01:57:37,320 >> YEAH. 3184 01:57:37,320 --> 01:57:39,456 THAT IS A REALLY GOOD QUESTION. 3185 01:57:39,456 --> 01:57:42,659 I WORKED WITH JEFF IN AN 3186 01:57:42,659 --> 01:57:43,726 INSTANCE WHERE WE WERE THINKING 3187 01:57:43,726 --> 01:57:44,227 ABOUT THIS. 3188 01:57:44,227 --> 01:57:47,130 I THINK ONE OF THE THINGS THAT I 3189 01:57:47,130 --> 01:57:48,431 ALWAYS ENCOURAGE MY CLIENTS IS 3190 01:57:48,431 --> 01:57:50,066 WE THINK ABOUT WHAT IS YOUR 3191 01:57:50,066 --> 01:57:51,334 IDEAL LABEL, RIGHT? 3192 01:57:51,334 --> 01:57:53,903 AND WHAT IS THAT FIRST 3193 01:57:53,903 --> 01:57:55,472 POPULATION YOU MIGHT BE YOUR 3194 01:57:55,472 --> 01:57:57,207 OPPORTUNITY FOR APPROVAL? 3195 01:57:57,207 --> 01:57:59,342 AND THEN WHAT WOULD BE THOSE 3196 01:57:59,342 --> 01:58:00,777 APPROVAL END POINTS? 3197 01:58:00,777 --> 01:58:02,579 I ALWAYS LIKE TO HAVE THE WORD 3198 01:58:02,579 --> 01:58:04,747 PLURAL SO YOU HAVE IN YOUR BACK 3199 01:58:04,747 --> 01:58:06,683 POCKET THESE ARE THE THREE 3200 01:58:06,683 --> 01:58:08,585 THINGS THAT COULD POTENTIALLY BE 3201 01:58:08,585 --> 01:58:14,624 THE END POINTS THAT WE USE FOR 3202 01:58:14,624 --> 01:58:14,891 APPROVAL. 3203 01:58:14,891 --> 01:58:17,360 IF THERE IS ANY WAY YOU CAN BAKE 3204 01:58:17,360 --> 01:58:20,964 THEM IN YOUR PHASE 1 TRIAL EVEN 3205 01:58:20,964 --> 01:58:23,132 IF IT EXPLORATORY TO FIGURE OUT 3206 01:58:23,132 --> 01:58:24,834 HOW TO GATHER DATA, TO ME, I 3207 01:58:24,834 --> 01:58:26,603 THINK THAT IS REALLY IMPORTANT. 3208 01:58:26,603 --> 01:58:28,605 DOES THAT ANSWER YOUR QUESTION? 3209 01:58:28,605 --> 01:58:29,172 >> YEAH. 3210 01:58:29,172 --> 01:58:31,140 IT CERTAINLY HELPS US HAVE THE 3211 01:58:31,140 --> 01:58:31,474 CONVERSATION. 3212 01:58:31,474 --> 01:58:33,610 IT SEEMS LIKE THIS IS GOING TO 3213 01:58:33,610 --> 01:58:36,379 BE AN ONGOING CONVERSATION FOR 3214 01:58:36,379 --> 01:58:40,183 OUR COMMUNITY. 3215 01:58:40,183 --> 01:58:42,919 ROBIN, JEN ASKED APPROPRIATELY, 3216 01:58:42,919 --> 01:58:46,756 CAN YOU TALK ABOUT PATIENT 3217 01:58:46,756 --> 01:58:53,096 RECRUITMENT GIVEN THE RARE 3218 01:58:53,096 --> 01:58:57,100 NATURE OF THESE DISEASES? 3219 01:58:57,100 --> 01:58:59,135 >> I DIDN'T GET INTO DETAIL, BUT 3220 01:58:59,135 --> 01:59:01,237 THERE IS A VERY COMPLICATED 3221 01:59:01,237 --> 01:59:02,805 STUDY FOR OUR COMMUNITY. 3222 01:59:02,805 --> 01:59:05,108 THERE WERE ANIMAL STUDY CONCERNS 3223 01:59:05,108 --> 01:59:06,943 ABOUT TEETH DEVELOPMENT, BONE 3224 01:59:06,943 --> 01:59:07,243 DEVELOPMENT. 3225 01:59:07,243 --> 01:59:10,547 SO THIS STUDY DESIGN INCLUDED 3226 01:59:10,547 --> 01:59:14,017 IMAGING OF THE TEETH, LONG BONE 3227 01:59:14,017 --> 01:59:16,953 MRIs AND SO IN ADDITION TO 3228 01:59:16,953 --> 01:59:19,923 HAVING A RARE COMMUNITY, IT 3229 01:59:19,923 --> 01:59:21,591 COMPLICATED A LOT OF VISITS. 3230 01:59:21,591 --> 01:59:24,027 MY HOPE IS THE NEXT STUDIES 3231 01:59:24,027 --> 01:59:25,662 AREN'T AS COMPLICATED. 3232 01:59:25,662 --> 01:59:26,996 I THINK THE COMMUNITY WAS A 3233 01:59:26,996 --> 01:59:28,164 LITTLE HESITANT. 3234 01:59:28,164 --> 01:59:30,733 THIS WAS A VERY SICK POPULATION 3235 01:59:30,733 --> 01:59:32,602 SO THESE PATIENTS WHO CAME 3236 01:59:32,602 --> 01:59:33,536 FORWARD CAME FORWARD BECAUSE 3237 01:59:33,536 --> 01:59:34,804 THEY WERE SICK. 3238 01:59:34,804 --> 01:59:36,973 THERE WERE STILL HESITATIONS TO 3239 01:59:36,973 --> 01:59:37,373 ENROLL. 3240 01:59:37,373 --> 01:59:39,142 I THINK THIS ALSO HAS GIVEN 3241 01:59:39,142 --> 01:59:40,410 HOPEFULLY SOME OF THE PATIENTS 3242 01:59:40,410 --> 01:59:46,549 CONFIDENCE THAT WE CAN DO THIS. 3243 01:59:46,549 --> 01:59:48,618 THE SPONSOR PAID FOR PATIENTS TO 3244 01:59:48,618 --> 01:59:50,453 FLY IN. 3245 01:59:50,453 --> 01:59:53,423 SO PATIENTS FLEW IN TO PRIMARY 3246 01:59:53,423 --> 01:59:55,024 STUDIES AND ALL OF THAT WAS PAID 3247 01:59:55,024 --> 01:59:57,427 FOR SO THEY COULD FLY IN EVEN 3248 01:59:57,427 --> 01:59:59,028 THOUGH IT WAS FREQUENT. 3249 01:59:59,028 --> 02:00:01,197 THE PATIENTS MADE COMMITMENTS TO 3250 02:00:01,197 --> 02:00:02,799 FLY INTO CENTERS DOING THIS 3251 02:00:02,799 --> 02:00:06,436 STUDY TO GET ACCESS TO THIS 3252 02:00:06,436 --> 02:00:06,836 DRUG. 3253 02:00:06,836 --> 02:00:08,972 SO SOME OF THIS INFORMATION THE 3254 02:00:08,972 --> 02:00:11,708 PATIENT ORGANIZATIONS WILL BE 3255 02:00:11,708 --> 02:00:13,776 HELPFUL TO UNDERSTAND. 3256 02:00:13,776 --> 02:00:15,545 I THINK HAVING RARE DISEASE HAVE 3257 02:00:15,545 --> 02:00:18,381 ACCESS TO STUDY CENTERS, IT WILL 3258 02:00:18,381 --> 02:00:20,149 TAKE ABILITY TO GET THOSE 3259 02:00:20,149 --> 02:00:21,851 PATIENTS TO THOSE CENTERS TO 3260 02:00:21,851 --> 02:00:27,890 PARTICIPATE. 3261 02:00:27,890 --> 02:00:29,692 >> RELATED TO PICKING OUR 3262 02:00:29,692 --> 02:00:32,128 PATIENTS FOR CLINICAL TRIALS, 3263 02:00:32,128 --> 02:00:34,597 QUESTION DIRECTED AT KAREN, BUT 3264 02:00:34,597 --> 02:00:35,965 IMPORTANT TO CONSIDER. 3265 02:00:35,965 --> 02:00:38,368 SO IS THAT PATIENT ENROLLMENT 3266 02:00:38,368 --> 02:00:40,303 CRITERIA TYPICALLY CO-DEFINED 3267 02:00:40,303 --> 02:00:41,504 BETWEEN INVESTIGATOR AND THE 3268 02:00:41,504 --> 02:00:41,738 COMPANY? 3269 02:00:41,738 --> 02:00:45,642 DOES THE INNOVATOR COMPANY 3270 02:00:45,642 --> 02:00:47,877 ENGAGE WITH THE PARTNERS AND 3271 02:00:47,877 --> 02:00:49,746 INVESTIGATOR ON AN ONGOING 3272 02:00:49,746 --> 02:00:50,146 BASIS? 3273 02:00:50,146 --> 02:00:51,648 HOW DOES THAT AGREEMENT COME 3274 02:00:51,648 --> 02:00:52,849 INTO PLAY FROM A CONTRACT 3275 02:00:52,849 --> 02:00:53,516 STANDPOINT? 3276 02:00:53,516 --> 02:00:54,550 >> YEAH. 3277 02:00:54,550 --> 02:00:56,686 I THINK FOR 3278 02:00:56,686 --> 02:00:57,654 INVESTIGATOR-INITIATED STUDIES, 3279 02:00:57,654 --> 02:01:00,189 IT IS GOING TO BE THE PROPOSAL 3280 02:01:00,189 --> 02:01:01,591 THAT COMES FROM THE INVESTIGATOR 3281 02:01:01,591 --> 02:01:02,725 SPONSOR. 3282 02:01:02,725 --> 02:01:04,961 AND SO AS LONG AS IT MAKES SENSE 3283 02:01:04,961 --> 02:01:08,498 TO THE COMPANY PROVIDING SUPPORT 3284 02:01:08,498 --> 02:01:10,099 THEN THAT'S GOING TO BE HOW IT 3285 02:01:10,099 --> 02:01:13,569 IS WRITTEN IN THE CONTRACT. 3286 02:01:13,569 --> 02:01:15,004 OBVI 3287 02:01:15,004 --> 02:01:16,572 OBVIOUSLY, IT IS A DIFFERENT 3288 02:01:16,572 --> 02:01:18,241 SITUATION FOR A 3289 02:01:18,241 --> 02:01:19,442 COMPANY-SPONSORED TRIAL SO A 3290 02:01:19,442 --> 02:01:21,144 COMPANY CAN MAKE THOSE 3291 02:01:21,144 --> 02:01:23,746 DETERMINATIONS WITH THE HELP OF 3292 02:01:23,746 --> 02:01:25,848 THOSE ADVISERS. 3293 02:01:25,848 --> 02:01:29,652 >> WE ALSO JUST TALKING ABOUT 3294 02:01:29,652 --> 02:01:32,755 CONTRACTS, DR. McCORMACK NOTED 3295 02:01:32,755 --> 02:01:34,724 THAT TRIALS AT THEIR FACILITIES 3296 02:01:34,724 --> 02:01:36,526 ARE RATE LIMITING STEP, THE 3297 02:01:36,526 --> 02:01:38,961 CONTRACTING SEEMS TO BE A RATE 3298 02:01:38,961 --> 02:01:40,496 LIMITING STEP AND LEGAL TEAM IS 3299 02:01:40,496 --> 02:01:42,999 STRETCHED PRETTY THIN ACROSS THE 3300 02:01:42,999 --> 02:01:43,299 UNIVERSITY. 3301 02:01:43,299 --> 02:01:46,803 SO ARE THERE PLACES THAT HAVE 3302 02:01:46,803 --> 02:01:48,404 DEDICATED LEGAL COUNSEL FOR 3303 02:01:48,404 --> 02:01:51,641 TRIALS IN PARTICULAR THAT YOU 3304 02:01:51,641 --> 02:01:52,475 KNOW OF? 3305 02:01:52,475 --> 02:01:55,078 ARE WE ABLE TO SHOW THAT IS 3306 02:01:55,078 --> 02:01:55,678 FINANCIALLY FEASIBLE FOR OUR 3307 02:01:55,678 --> 02:01:56,813 INSTITUTIONS? 3308 02:01:56,813 --> 02:01:58,114 >> YEAH. 3309 02:01:58,114 --> 02:01:59,782 IT'S TYPICALLY THERE'S A 3310 02:01:59,782 --> 02:02:01,818 CLINICAL TRIALS OFFICE THAT 3311 02:02:01,818 --> 02:02:04,153 HANDLES CONTRACTING AT 3312 02:02:04,153 --> 02:02:04,654 INSTITUTIONS. 3313 02:02:04,654 --> 02:02:06,456 IT IS A VERY DIFFERENT SET OF 3314 02:02:06,456 --> 02:02:08,057 PRIORITIES I THINK FOR A COMPANY 3315 02:02:08,057 --> 02:02:09,759 LIKE MINE IT IS SORT OF WHAT IS 3316 02:02:09,759 --> 02:02:11,994 IMPORTANT TO THE COMPANY, RIGHT? 3317 02:02:11,994 --> 02:02:13,896 AND THEN, YOU KNOW, WE ARE SORT 3318 02:02:13,896 --> 02:02:17,300 OF MOTIVATED A LITTLE BIT 3319 02:02:17,300 --> 02:02:19,068 DIFFERENTLY TO MAYBE PULL AN 3320 02:02:19,068 --> 02:02:20,369 ALL-NIGHTER IF YOU HAVE TO, BUT 3321 02:02:20,369 --> 02:02:21,838 THAT IS NOT THE CULTURE AT 3322 02:02:21,838 --> 02:02:23,740 UNIVERSITY OFFICES. 3323 02:02:23,740 --> 02:02:25,341 SO I THINK WHAT UNIVERSITY 3324 02:02:25,341 --> 02:02:27,110 OFFICES IN MY EXPERIENCE REACT 3325 02:02:27,110 --> 02:02:29,912 TO IS THE SQUEAKY WHEEL, RIGHT? 3326 02:02:29,912 --> 02:02:31,214 THE INVESTIGATOR WHO IS MAKING 3327 02:02:31,214 --> 02:02:33,015 THE CALLS, WHO IS REALLY 3328 02:02:33,015 --> 02:02:39,088 ADVOCATING FOR THE STUDY. 3329 02:02:39,088 --> 02:02:40,757 WHEN WE NEED SOMETHING MOVED 3330 02:02:40,757 --> 02:02:43,292 ALONG, WE GET THE INVESTIGATOR 3331 02:02:43,292 --> 02:02:44,727 INVOLVED BECAUSE THE UNIVERSITY 3332 02:02:44,727 --> 02:02:45,595 LEGAL OFFICE DOES NOT CARE WHAT 3333 02:02:45,595 --> 02:02:47,497 I HAVE TO SAY. 3334 02:02:47,497 --> 02:02:49,599 THEY CARE MORE ABOUT THEIR OWN 3335 02:02:49,599 --> 02:02:53,703 INVESTIGATORS. 3336 02:02:53,703 --> 02:02:55,738 >> ONE QUESTION THAT REALLY IS 3337 02:02:55,738 --> 02:02:59,342 OPEN TO ANYBODY, REALLY, IN THE 3338 02:02:59,342 --> 02:03:00,643 ENTIRE SPACE. 3339 02:03:00,643 --> 02:03:03,246 SO IF YOU HAVE -- TALK ABOUT 3340 02:03:03,246 --> 02:03:07,183 THAT PROCESS OF HAVING AN 3341 02:03:07,183 --> 02:03:08,985 INVESTIGATOR-INITIATED IND AND 3342 02:03:08,985 --> 02:03:10,653 IF THERE IS COMMERCIAL INTEREST 3343 02:03:10,653 --> 02:03:12,288 HOW THAT BECOMES COMMERCIALLY 3344 02:03:12,288 --> 02:03:15,258 SUPPORTED IND IF A COMPANY 3345 02:03:15,258 --> 02:03:17,860 BECOMES INTERESTED MID TRIAL. 3346 02:03:17,860 --> 02:03:18,961 LIKE YOU ARE ALREADY DOING THE 3347 02:03:18,961 --> 02:03:20,830 BASIC WORK. 3348 02:03:20,830 --> 02:03:22,899 >> I CAN JUST COMMENT ON ONE 3349 02:03:22,899 --> 02:03:23,166 SCENARIO. 3350 02:03:23,166 --> 02:03:26,402 I HAVE SEEN IN THE PAST AN 3351 02:03:26,402 --> 02:03:30,406 INVESTIGATOR INITIATED PROTOCOL 3352 02:03:30,406 --> 02:03:33,009 BECOME A COMPANY-SPONSORED 3353 02:03:33,009 --> 02:03:33,342 PROTOCOL. 3354 02:03:33,342 --> 02:03:34,844 IT WAS SET UP ALMOST LIKE A 3355 02:03:34,844 --> 02:03:35,444 PURCHASE. 3356 02:03:35,444 --> 02:03:37,146 PAYING FOR THE SERVICES OF 3357 02:03:37,146 --> 02:03:37,847 PROTOCOL DEVELOPMENT. 3358 02:03:37,847 --> 02:03:39,315 I HAVE SEEN THAT HAPPEN. 3359 02:03:39,315 --> 02:03:40,516 AND IT IS POSSIBLE. 3360 02:03:40,516 --> 02:03:42,351 YOU CAN DO ANYTHING THROUGH 3361 02:03:42,351 --> 02:03:44,720 CONTRACTING, I ALWAYS SAY. 3362 02:03:44,720 --> 02:03:46,923 THAT WAS JUST MY OWN EXPERIENCE. 3363 02:03:46,923 --> 02:03:48,891 >> SO IN MY EXPERIENCE, I HAVE 3364 02:03:48,891 --> 02:03:51,127 WORKED IN A COUPLE OF SCENARIOS 3365 02:03:51,127 --> 02:03:57,233 AND RECENTLY HAD A CLIENT THAT 3366 02:03:57,233 --> 02:04:00,436 WAS I.T. AND INSTEAD OF -- THEY 3367 02:04:00,436 --> 02:04:02,238 DID A COMPANY SPINOFF TO START 3368 02:04:02,238 --> 02:04:04,440 THE PHASE 2 TRIAL AND WE JUST 3369 02:04:04,440 --> 02:04:08,044 DID A FRESH NEW IND TO KEEP 3370 02:04:08,044 --> 02:04:09,045 EVERYTHING CLEAN AND CROSS 3371 02:04:09,045 --> 02:04:10,479 REFERENCED THE IND. 3372 02:04:10,479 --> 02:04:11,914 CAN'T SPEAK TO THE CONTRACTING 3373 02:04:11,914 --> 02:04:12,114 SIDE. 3374 02:04:12,114 --> 02:04:13,516 THE OTHER STRATEGY I HAVE USED 3375 02:04:13,516 --> 02:04:18,321 THAT HAS WORKED REALLY WELL IS A 3376 02:04:18,321 --> 02:04:21,591 HYBRID IIT, MAYBE THE DRUG 3377 02:04:21,591 --> 02:04:23,192 DEVELOPER INTERESTED MAY HOLD 3378 02:04:23,192 --> 02:04:26,028 THE IND OR MAYBE THEY ARE MORE 3379 02:04:26,028 --> 02:04:30,333 HEAVILY INVOLVE SOD THE ELEMENT 3380 02:04:30,333 --> 02:04:33,169 OF DATA HAVING IT IN -- FORMAT 3381 02:04:33,169 --> 02:04:35,705 AND ALL OF THAT, SAFETY, SOME OF 3382 02:04:35,705 --> 02:04:36,739 THOSE ELEMENTS CAN BE ROLLED IN. 3383 02:04:36,739 --> 02:04:40,109 I WORKED WITH A CLIENT WHERE WE 3384 02:04:40,109 --> 02:04:44,513 DID A HYBRID IIT. 3385 02:04:44,513 --> 02:04:45,948 WE CONVERTED THAT PHASE 1 TRIAL 3386 02:04:45,948 --> 02:04:47,884 INTO A PIVOTAL TRIAL BECAUSE IT 3387 02:04:47,884 --> 02:04:49,919 WAS SO -- WE HAD 100% RESPONSE 3388 02:04:49,919 --> 02:04:52,688 RATE OF THE PATIENTS. 3389 02:04:52,688 --> 02:04:56,225 AND IT WAS A RARE DISEASE. 3390 02:04:56,225 --> 02:04:57,426 SO I GUESS MAYBE MY POINT IS 3391 02:04:57,426 --> 02:04:59,195 WHEN I TALK TO MY CLIENTS THERE 3392 02:04:59,195 --> 02:05:01,797 ARE OPTIONS AND IT IS ALWAYS 3393 02:05:01,797 --> 02:05:03,699 GOOD TO KIND OF PUT YOUR STICKY 3394 02:05:03,699 --> 02:05:05,601 NOTE FROM THE WALL AND SEE WHAT 3395 02:05:05,601 --> 02:05:07,003 THE OPTIONS ARE TO SEE WHAT 3396 02:05:07,003 --> 02:05:08,437 STRATEGY IS GOING TO WORK THE 3397 02:05:08,437 --> 02:05:08,804 BEST. 3398 02:05:08,804 --> 02:05:11,407 >> THAT IS ACTUALLY A REALLY 3399 02:05:11,407 --> 02:05:12,742 GOOD POINT, DORIS. 3400 02:05:12,742 --> 02:05:16,145 WE HAVE OTHER OPTIONS OTHER THAN 3401 02:05:16,145 --> 02:05:19,649 COMPANY-SPONSORED TRIALS AND 3402 02:05:19,649 --> 02:05:20,549 INVESTIGATOR-INITIATED TRIALS. 3403 02:05:20,549 --> 02:05:22,351 WE CALL IT COLLABORATIVE. 3404 02:05:22,351 --> 02:05:23,853 YOU CAN CALL IT WHATEVER YOU 3405 02:05:23,853 --> 02:05:24,954 WANT IT LOOKS IN BETWEEN. 3406 02:05:24,954 --> 02:05:26,689 IT IS FOR THE PARTIES TO DECIDE 3407 02:05:26,689 --> 02:05:28,925 WHAT THE ROLES AND 3408 02:05:28,925 --> 02:05:29,859 RESPONSIBILITIES ARE. 3409 02:05:29,859 --> 02:05:30,726 WE WOULD CALL THAT COLLABORATIVE 3410 02:05:30,726 --> 02:05:34,063 RESEARCH. 3411 02:05:34,063 --> 02:05:36,232 >> I DEFINITELY WANT TO GET BACK 3412 02:05:36,232 --> 02:05:38,200 TO TALKING SOME ABOUT END POINTS 3413 02:05:38,200 --> 02:05:39,135 BECAUSE WE HAVE SOME QUESTIONS 3414 02:05:39,135 --> 02:05:40,202 IN THE CHAT ABOUT THAT. 3415 02:05:40,202 --> 02:05:42,939 I ALSO WANT TO GO BACK TO THAT 3416 02:05:42,939 --> 02:05:45,474 FIRST TALK THAT WE HAD, JEN, YOU 3417 02:05:45,474 --> 02:05:47,510 TALKED ABOUT SOME OF THE 3418 02:05:47,510 --> 02:05:48,711 BARRIERS YOU SEE FROM BRINGING 3419 02:05:48,711 --> 02:05:50,313 TREATMENTS TO INFANTS. 3420 02:05:50,313 --> 02:05:51,447 AND YOU HAVE THIS WONDERFUL LIST 3421 02:05:51,447 --> 02:05:52,782 OF CONSIDERATIONS WE AS A 3422 02:05:52,782 --> 02:05:55,651 COMMUNITY NEED TO THINK ABOUT TO 3423 02:05:55,651 --> 02:05:57,420 MAKE THIS HAPPEN. 3424 02:05:57,420 --> 02:06:00,856 INCLUDING PROVIDER EDUCATION, 3425 02:06:00,856 --> 02:06:03,826 ACCESS TO GENETIC TESTING. 3426 02:06:03,826 --> 02:06:06,329 HOW DO WE GET PEOPLE TO THE 3427 02:06:06,329 --> 02:06:10,099 CENTERS OF EXPERTISE AND 3428 02:06:10,099 --> 02:06:13,736 COLLABORATE WITH NEONATOLOGY. 3429 02:06:13,736 --> 02:06:16,906 IF I HAD TO FORCE YOU TO PICK 3430 02:06:16,906 --> 02:06:18,174 ONE OR TWO THINGS OUR COMMUNITY 3431 02:06:18,174 --> 02:06:20,176 SHOULD BE FOCUSED ON, WHAT IS 3432 02:06:20,176 --> 02:06:20,943 THAT SPACE FOR YOU? 3433 02:06:20,943 --> 02:06:22,278 >> IT IS A GREAT QUESTION. 3434 02:06:22,278 --> 02:06:25,114 I THINK ACCESS TO GENETIC 3435 02:06:25,114 --> 02:06:26,849 TESTING WOULD BE THE TOP 3436 02:06:26,849 --> 02:06:27,483 PRIORITY. 3437 02:06:27,483 --> 02:06:35,925 WE ARE WORKING ON A NEW AMERICAN 3438 02:06:35,925 --> 02:06:36,258 TH 3439 02:06:36,258 --> 02:06:37,159 THORACI STUDY STATEMENT. 3440 02:06:37,159 --> 02:06:40,262 WITH THAT WE ARE HOPEFUL THERE 3441 02:06:40,262 --> 02:06:41,731 WILL BE RECOMMENDATIONS ABOUT 3442 02:06:41,731 --> 02:06:43,766 GENETIC TESTING IN INFANTS AND 3443 02:06:43,766 --> 02:06:44,900 CHILDREN. 3444 02:06:44,900 --> 02:06:46,068 HOPEFULLY GETTING THE DIAGNOSES 3445 02:06:46,068 --> 02:06:46,402 SOONER. 3446 02:06:46,402 --> 02:06:47,837 WAS THE DIAGNOSIS IS MADE AND 3447 02:06:47,837 --> 02:06:49,839 SOMETIMES THAT IS COMPLICATED, 3448 02:06:49,839 --> 02:06:52,508 BUT IN GENERAL, PEOPLE CAN REACH 3449 02:06:52,508 --> 02:06:54,276 OUT TO THEIR PULMONOLOGIST AT 3450 02:06:54,276 --> 02:06:57,346 THEIR CENTER WHO MAYBE IF THEY 3451 02:06:57,346 --> 02:06:59,315 ARE NOT AN EXPERT IN ILD CAN 3452 02:06:59,315 --> 02:07:01,283 CONNECT TO SOMEONE WHO IS. 3453 02:07:01,283 --> 02:07:03,019 CONTINUING TO HAVE A PRESENCE AT 3454 02:07:03,019 --> 02:07:04,787 NATIONAL MEETINGS, CONTINUING TO 3455 02:07:04,787 --> 02:07:07,590 TALK ABOUT DIAGNOSIS AND 3456 02:07:07,590 --> 02:07:09,925 UPCOMING THERAPIES TO GENERATE 3457 02:07:09,925 --> 02:07:11,027 AWARENESS AND RECOGNITION I 3458 02:07:11,027 --> 02:07:15,664 THINK IS REALLY IMPORTANT. 3459 02:07:15,664 --> 02:07:17,700 >> IF WE CAN'T FIND THE PATIENTS 3460 02:07:17,700 --> 02:07:19,602 WE CAN'T HELP THE PATIENTS, 3461 02:07:19,602 --> 02:07:19,835 RIGHT? 3462 02:07:19,835 --> 02:07:20,436 >> CORRECT. 3463 02:07:20,436 --> 02:07:22,638 >> I JUST WANTED TO ADD BECAUSE 3464 02:07:22,638 --> 02:07:26,075 JEN IS TALKING ABOUT THOSE 3465 02:07:26,075 --> 02:07:29,211 YOUNGER PEDIATRIC PATIENTS. 3466 02:07:29,211 --> 02:07:30,913 THINKING ABOUT VULNERABLE GROUPS 3467 02:07:30,913 --> 02:07:33,749 IN THE PEDIATRIC POPULATION 3468 02:07:33,749 --> 02:07:36,685 CREATES A SPECIAL TASK RELATED 3469 02:07:36,685 --> 02:07:38,487 TO END POINTS. 3470 02:07:38,487 --> 02:07:43,826 THE REGULATORY AGENCIES LIKE 3471 02:07:43,826 --> 02:07:47,296 TRADITIONAL END POINTS, 3472 02:07:47,296 --> 02:07:49,298 PULMONARY FUNCTIONING TEST, 3473 02:07:49,298 --> 02:07:50,299 CAPACITY IN FIBROSIS. 3474 02:07:50,299 --> 02:07:52,968 I WOULD SAY WE DO HAVE SOME 3475 02:07:52,968 --> 02:07:55,571 EVOLVING DATA THAT IN GROWING 3476 02:07:55,571 --> 02:07:58,007 CHILDREN WITH LUNG FUNCTION THAT 3477 02:07:58,007 --> 02:07:59,875 PULMONARY FUNCTIONS MAY NOT BE 3478 02:07:59,875 --> 02:08:02,711 THE BEST INDICATOR OF ADVANCING 3479 02:08:02,711 --> 02:08:02,978 DISEASE. 3480 02:08:02,978 --> 02:08:09,618 IT MAY BE IMAGING WHERE FIBROSIS 3481 02:08:09,618 --> 02:08:11,053 IS EVOLVING. 3482 02:08:11,053 --> 02:08:13,122 SO THERE IS A LOT WE NEED TO 3483 02:08:13,122 --> 02:08:15,024 LEARN ABOUT KIDS. 3484 02:08:15,024 --> 02:08:19,228 WE CAN FIT IN WITH TRADITIONAL 3485 02:08:19,228 --> 02:08:19,595 APPROACHES. 3486 02:08:19,595 --> 02:08:23,265 I'M HOPEFUL WE CAN THINK 3487 02:08:23,265 --> 02:08:23,632 BROADER. 3488 02:08:23,632 --> 02:08:26,302 YOU SAW THE FAMILIAR SCORE, A 3489 02:08:26,302 --> 02:08:29,772 BIG WIN IN THE EUROPEAN. 3490 02:08:29,772 --> 02:08:34,043 THE FAN SCORE HAD TO DO WITH 3491 02:08:34,043 --> 02:08:36,579 OXYGEN. 3492 02:08:36,579 --> 02:08:40,783 PHYSIOLOGIC MEASUREMENTS IN 3493 02:08:40,783 --> 02:08:44,420 CHILDREN, JEN IS A 3494 02:08:44,420 --> 02:08:45,254 NEONATOLOGIST. 3495 02:08:45,254 --> 02:08:47,490 THAT TESTING IS NOT HAPPENING. 3496 02:08:47,490 --> 02:08:50,025 HOW DO WE BEGIN TO VALIDATE 3497 02:08:50,025 --> 02:08:52,528 THOSE IN CLINICAL TRIALS? 3498 02:08:52,528 --> 02:08:54,597 WE ARE GOING TO NEED THAT. 3499 02:08:54,597 --> 02:08:56,665 THERE ARE NEW DEVICES AND NEW 3500 02:08:56,665 --> 02:08:58,901 WAYS TO THINK ABOUT THIS. 3501 02:08:58,901 --> 02:09:00,369 THOSE QUESTIONS NEED TO BE 3502 02:09:00,369 --> 02:09:01,203 ASKED. 3503 02:09:01,203 --> 02:09:05,474 WE NEED TO BE MORE ASSERTIVE FOR 3504 02:09:05,474 --> 02:09:07,943 THE PEDIATRIC GROUP. 3505 02:09:07,943 --> 02:09:09,979 MAYBE INDEPENDENT INVESTIGATOR 3506 02:09:09,979 --> 02:09:14,750 TRIALS TO ASSESS THAT AS AN 3507 02:09:14,750 --> 02:09:17,620 AJUNCTION TO SOME OF THE BIGGER 3508 02:09:17,620 --> 02:09:20,356 TRIALS RUNNING FORWARD. 3509 02:09:20,356 --> 02:09:21,590 I FEEL STRONGLY WE NEED TO DO 3510 02:09:21,590 --> 02:09:29,565 THAT. 3511 02:09:29,565 --> 02:09:32,401 I THINK DR. KAITH POSTED SHE 3512 02:09:32,401 --> 02:09:35,037 WORKS IN THE SPACE SO LOTS OF 3513 02:09:35,037 --> 02:09:37,239 INTEREST IN VALIDATING NEW 3514 02:09:37,239 --> 02:09:37,473 STUDIES. 3515 02:09:37,473 --> 02:09:40,643 ARE THERE END POINTS BEST 3516 02:09:40,643 --> 02:09:41,644 TRANSLATE TO PEDIATRIC 3517 02:09:41,644 --> 02:09:41,944 POPULATIONS. 3518 02:09:41,944 --> 02:09:44,813 IT IS MORE OF A PHYSIOLOGIC 3519 02:09:44,813 --> 02:09:46,315 RESPONSE OR MEASURE TO DISEASE. 3520 02:09:46,315 --> 02:09:48,083 IS THERE ANYTHING YOU GUYS HAVE 3521 02:09:48,083 --> 02:09:49,985 SEEN IN THE ADULT LITERATURE YOU 3522 02:09:49,985 --> 02:09:51,954 THINK WE COULD WORK TOGETHER ON 3523 02:09:51,954 --> 02:09:54,623 OR WAYS TO KIND OF, AGAIN, 3524 02:09:54,623 --> 02:09:55,925 BORROW FROM THEIR SPACE THAT 3525 02:09:55,925 --> 02:10:05,301 MIGHT WORK IN OUR PATIENTS? 3526 02:10:05,301 --> 02:10:07,436 THIS IS ONE, TOO, I THINK 3527 02:10:07,436 --> 02:10:08,771 ANYBODY CAN UNMUTE THEMSELVES AT 3528 02:10:08,771 --> 02:10:09,405 THIS POINT. 3529 02:10:09,405 --> 02:10:10,873 WE HAVE A FEW MINUTES. 3530 02:10:10,873 --> 02:10:13,342 SO IF THERE ARE PEOPLE WHO 3531 02:10:13,342 --> 02:10:17,613 WANTED TO ADD INTO THIS 3532 02:10:17,613 --> 02:10:20,049 DISCUSSION VERBALLY, I'M OKAY 3533 02:10:20,049 --> 02:10:22,117 WITH THAT THE OTHER PEOPLE HAVE 3534 02:10:22,117 --> 02:10:24,653 THOUGHTS ON THIS END POINT 3535 02:10:24,653 --> 02:10:25,154 DISCUSSION. 3536 02:10:25,154 --> 02:10:28,857 >> I WILL ADD PULSE OX DLERKS 3537 02:10:28,857 --> 02:10:30,459 METRY RESPIRATORY RATE, WHICH IS 3538 02:10:30,459 --> 02:10:33,329 A HARD ONE TO MEASURE 3539 02:10:33,329 --> 02:10:35,631 ACCURATELY, BUT SOME DEVICES ARE 3540 02:10:35,631 --> 02:10:41,337 DOING THIS IN A VALIDATED WAY. 3541 02:10:41,337 --> 02:10:43,539 POTENTIALLY MOVEMENT OR KIND OF 3542 02:10:43,539 --> 02:10:45,407 MOVEMENT-BASED IS A LOT OF 3543 02:10:45,407 --> 02:10:49,111 DEVICES NOW THAT ARE LOOKING AT 3544 02:10:49,111 --> 02:10:49,511 MOVEMENT. 3545 02:10:49,511 --> 02:10:53,382 THAT THESE ARE ALL POTENTIAL 3546 02:10:53,382 --> 02:10:54,550 INDICATIONS OF PULMONARY HEALTH 3547 02:10:54,550 --> 02:10:55,584 OR HEALTH. 3548 02:10:55,584 --> 02:10:59,788 THEY NEED TO BE EXPLORED. 3549 02:10:59,788 --> 02:11:03,425 I'M HOPING NHLBI WILL HELP US. 3550 02:11:03,425 --> 02:11:05,127 DR. WAMBACH, WHAT DO YOU THINK 3551 02:11:05,127 --> 02:11:06,462 OF THAT FOR YOUR BABIES? 3552 02:11:06,462 --> 02:11:10,633 WE ARE NOT DOING PFTs 3553 02:11:10,633 --> 02:11:11,400 NECESSARILY ON BABIES. 3554 02:11:11,400 --> 02:11:13,435 WE DON'T HAVE A WAY TO DO THAT? 3555 02:11:13,435 --> 02:11:16,305 >> WE CAN LOOK AT MARKERS OF 3556 02:11:16,305 --> 02:11:22,711 VENTILATION, OXYGENATION, 3557 02:11:22,711 --> 02:11:24,213 VENTILATOR CHANGING OVER TIME, 3558 02:11:24,213 --> 02:11:27,916 LOOPS ON VENTILATORS. 3559 02:11:27,916 --> 02:11:28,784 BIOMARKERS WOULD BE TERRIFIC. 3560 02:11:28,784 --> 02:11:37,092 WE LOOKED AT THE PHOSPHOLIPID 3561 02:11:37,092 --> 02:11:37,326 CONTENT. 3562 02:11:37,326 --> 02:11:41,697 DR. ZIYADI TALKED ABOUT 3563 02:11:41,697 --> 02:11:43,432 YESTERDAY WITH PATIENTS FROM 3564 02:11:43,432 --> 02:11:45,901 CYSTIC FIBROSIS COULD WE 3565 02:11:45,901 --> 02:11:47,136 ESTABLISH BIOMARKERS FOR THESE 3566 02:11:47,136 --> 02:11:47,670 CONDITIONS. 3567 02:11:47,670 --> 02:11:49,605 ROBIN, I KNOW YOU HAVE WORKED IN 3568 02:11:49,605 --> 02:11:51,774 THAT SPACE A BIT WITH CHILDREN 3569 02:11:51,774 --> 02:11:53,609 WITH ILD SO MAYBE YOU WANT TO 3570 02:11:53,609 --> 02:11:58,614 COMMENT ON THAT. 3571 02:11:58,614 --> 02:12:01,116 >> I THINK THE BIOMARKER WORLD 3572 02:12:01,116 --> 02:12:02,985 DEVELOPMENT IS A TOUGH ONE. 3573 02:12:02,985 --> 02:12:04,353 PEOPLE HAVE BEEN DOING WORK FOR 3574 02:12:04,353 --> 02:12:05,821 A LONG TIME AND TRYING TO 3575 02:12:05,821 --> 02:12:07,089 VALIDATE A BIOMARKER. 3576 02:12:07,089 --> 02:12:13,495 I THINK DR. McCORMACK HAD SOME 3577 02:12:13,495 --> 02:12:16,031 EXCELLENT SUCCESS IN LAMB 3578 02:12:16,031 --> 02:12:17,533 THINKING ABOUT BIOMARKERS. 3579 02:12:17,533 --> 02:12:18,334 IT IS IMPORTANT WORK. 3580 02:12:18,334 --> 02:12:20,336 IT IS WORK THAT TAKES A LONG 3581 02:12:20,336 --> 02:12:20,569 TIME. 3582 02:12:20,569 --> 02:12:22,504 SO THAT IS WHY I LIKE TO THINK 3583 02:12:22,504 --> 02:12:24,273 ABOUT BIOMARKERS THAT CAN 3584 02:12:24,273 --> 02:12:26,108 GENERALIZE ACROSS MULTIPLE 3585 02:12:26,108 --> 02:12:26,608 DISEASES. 3586 02:12:26,608 --> 02:12:30,045 WE TAKE CARE OF KIDS WITH MANY 3587 02:12:30,045 --> 02:12:32,247 RARE DISEASES AND HOW DO WE 3588 02:12:32,247 --> 02:12:33,549 CREATE GENERALIZABLE APPROACHES 3589 02:12:33,549 --> 02:12:35,517 TO GET THE TREATMENT TO THE MORE 3590 02:12:35,517 --> 02:12:38,821 PATIENTS THE FASTEST? 3591 02:12:38,821 --> 02:12:40,856 WE HAVE PEOPLE ON THIS LINE WHO 3592 02:12:40,856 --> 02:12:42,091 CAN TALK ABOUT THAT. 3593 02:12:42,091 --> 02:12:45,327 >> JEN, TO YOUR POINT ON 3594 02:12:45,327 --> 02:12:46,729 BIOMARKERS, I'M SORRY, MY CAMERA 3595 02:12:46,729 --> 02:12:50,265 IS A LITTLE WEIRD, BUT, YEAH, WE 3596 02:12:50,265 --> 02:12:53,102 THINK DEVELOPING THOSE IS A GOOD 3597 02:12:53,102 --> 02:12:53,502 IDEA. 3598 02:12:53,502 --> 02:12:55,204 THERE IS DEFINITELY GOOD 3599 02:12:55,204 --> 02:12:56,739 EVIDENCE THE BLOOD BIOMARKERS 3600 02:12:56,739 --> 02:12:58,407 WILL BE USEFUL. 3601 02:12:58,407 --> 02:13:00,376 THE PLATFORM YOU USE IS 3602 02:13:00,376 --> 02:13:00,642 IMPORTANT. 3603 02:13:00,642 --> 02:13:02,244 THE NUMBER OF BIOMARKERS WILL BE 3604 02:13:02,244 --> 02:13:02,745 IMPORTANT. 3605 02:13:02,745 --> 02:13:04,380 FOR THE LUNG AND LUNG FUNCTION, 3606 02:13:04,380 --> 02:13:09,551 IT IS MORE COMPLEX THAN OTHER 3607 02:13:09,551 --> 02:13:12,388 SIMPLER PHYSIOLOGIC OUTCOMES. 3608 02:13:12,388 --> 02:13:15,157 I THINK YOU NEED MORE BIOMARKERS 3609 02:13:15,157 --> 02:13:16,125 THAN A HANDFUL. 3610 02:13:16,125 --> 02:13:18,227 WE ARE TRYING TO DEVELOP A PANEL 3611 02:13:18,227 --> 02:13:19,862 OF HUNDREDS OF BIOMARKERS IN THE 3612 02:13:19,862 --> 02:13:21,397 BLOOD WHICH IS COMPLEX BECAUSE 3613 02:13:21,397 --> 02:13:23,599 YOU HAVE TO VALIDATE IN SOMEONE. 3614 02:13:23,599 --> 02:13:27,503 LIKE ROBIN WAS MENTIONING WE 3615 02:13:27,503 --> 02:13:30,606 HAVE AN NIH GRANT AN RO1 ON 3616 02:13:30,606 --> 02:13:35,244 LOOKING AT HOW BIOMARKERS AND 3617 02:13:35,244 --> 02:13:39,415 IMAGING, HOW THEY BEHAVE 3618 02:13:39,415 --> 02:13:41,083 COMPARED WITH LUNG FUNCTION 3619 02:13:41,083 --> 02:13:42,651 MEASUREMENTS AND DEFINITELY PFTs 3620 02:13:42,651 --> 02:13:45,454 CAN BE STABLE OR UNCHANGED WHEN 3621 02:13:45,454 --> 02:13:47,856 YOU SEE DRASTIC DIFFERENCES IN 3622 02:13:47,856 --> 02:13:52,094 IMAGING WITH GAS TRAPPING, ZENON 3623 02:13:52,094 --> 02:13:55,964 IMAGING OR BIOMARKERINGS, 3624 02:13:55,964 --> 02:13:59,368 BIOMARKERINS WE HAVE EVIDENCE TY 3625 02:13:59,368 --> 02:14:01,170 ARE MORE SENSITIVE. 3626 02:14:01,170 --> 02:14:02,271 BIOMARKERS MAY COME EARLIER. 3627 02:14:02,271 --> 02:14:04,573 THEY NEED A LOT OF VALIDATION. 3628 02:14:04,573 --> 02:14:06,542 THEY ARE NOT EASY TO DEVELOP. 3629 02:14:06,542 --> 02:14:11,713 IT IS A COOL TOOL, BUT IT DOES 3630 02:14:11,713 --> 02:14:13,415 TAKE A LONG TIME. 3631 02:14:13,415 --> 02:14:15,884 IT WOULD BE GOOD IF THERE WERE 3632 02:14:15,884 --> 02:14:17,753 MORE FOLKS WORKING ON IT. 3633 02:14:17,753 --> 02:14:19,488 IT IS NOT AN AREA PEOPLE WANT TO 3634 02:14:19,488 --> 02:14:24,159 SPEND TIME ON. 3635 02:14:24,159 --> 02:14:28,363 >> THANK YOU FOR THAT. 3636 02:14:28,363 --> 02:14:31,467 WELL, WE ARE COMING UP TO THE 3637 02:14:31,467 --> 02:14:33,302 END OF OUR TIME HERE. 3638 02:14:33,302 --> 02:14:35,537 ANY FINAL COMMENTS FROM YOU 3639 02:14:35,537 --> 02:14:36,004 GUYS? 3640 02:14:36,004 --> 02:14:37,105 THANK YOU SO MUCH FOR GIVING 3641 02:14:37,105 --> 02:14:38,173 YOUR TIME AND SHARING YOUR 3642 02:14:38,173 --> 02:14:40,442 EXPERTISE. 3643 02:14:40,442 --> 02:14:42,511 THERE WILL BE MORE CONVERSATION 3644 02:14:42,511 --> 02:14:44,880 CERTAINLY THIS AFTERNOON. 3645 02:14:44,880 --> 02:14:46,482 HOPEFULLY YOU CAN JOIN BREAKOUT 3646 02:14:46,482 --> 02:14:48,584 SESSIONS AND HELP US THINK ABOUT 3647 02:14:48,584 --> 02:14:52,521 HOW WE BEST SUPPORT THIS. 3648 02:14:52,521 --> 02:14:53,989 >> THANK YOU ALL. 3649 02:14:53,989 --> 02:14:56,725 THIS WILL CLOSE OUT SESSION FOUR 3650 02:14:56,725 --> 02:14:58,861 AND RIGHT NOW ON OUR AGENDA WE 3651 02:14:58,861 --> 02:15:00,496 HAVE A LUNCH BREAK SCHEDULED 3652 02:15:00,496 --> 02:15:04,900 FROM 11:45 TO 12:15 P.M. EASTERN 3653 02:15:04,900 --> 02:15:05,100 TIME. 3654 02:15:05,100 --> 02:15:09,438 THEN WE WILL RETURN FOR OUR LAST 3655 02:15:09,438 --> 02:15:12,808 SCIENTIFIC SESSION, SUCCESS 3656 02:15:12,808 --> 02:15:14,167 STORIES FROM THE LUNG COMMUNITY. 3657 02:15:14,167 --> 02:15:16,402 I WILL BE CO-MODERATING THIS 3658 02:15:16,402 --> 02:15:19,739 SESSION WITH DR. AMY HAJARI 3659 02:15:19,739 --> 02:15:22,208 CASE, NEXT SLIDE, WHO IS THE 3660 02:15:22,208 --> 02:15:25,178 CHIEF MEDICAL OFFICER OF THE 3661 02:15:25,178 --> 02:15:25,778 PULMONARY FIBROSIS FOUNDATION 3662 02:15:25,778 --> 02:15:28,982 WHOSE MISSION IS TO SUPPORT 3663 02:15:28,982 --> 02:15:30,850 PULMONARY FIBROSIS RESEARCH, 3664 02:15:30,850 --> 02:15:32,018 EDUCATION AND ADVOCACY, SHE'S 3665 02:15:32,018 --> 02:15:33,286 ALSO A PRACTICING PHYSICIAN AND 3666 02:15:33,286 --> 02:15:35,688 SERVES AS THE DIRECTOR OF THE 3667 02:15:35,688 --> 02:15:37,957 ADVANCED LUNG DISEASE PROGRAM 3668 02:15:37,957 --> 02:15:47,567 AND MEDICAL DIRECTOR FOR 3669 02:15:47,567 --> 02:15:53,006 PULMONARY PIEDMONT HEALTHCARE, 3670 02:15:53,006 --> 02:15:54,507 AND THE DOCTOR WILL PROVIDE US 3671 02:15:54,507 --> 02:15:55,842 WITH INFORMATION FROM HER WORK 3672 02:15:55,842 --> 02:15:56,042 LATER. 3673 02:15:56,042 --> 02:15:56,342 NEXT SLIDE. 3674 02:15:56,342 --> 02:15:58,278 I WANT TO BRIEFLY INTRODUCE OUR 3675 02:15:58,278 --> 02:15:59,546 SPEAKERS BEFORE WE BEGIN THE 3676 02:15:59,546 --> 02:16:01,314 SESSION, AS A REMINDER, YOU CAN 3677 02:16:01,314 --> 02:16:03,416 POST YOUR QUESTIONS IN THE Q&A 3678 02:16:03,416 --> 02:16:04,617 TOOL AT ANY TIME DURING THIS 3679 02:16:04,617 --> 02:16:06,019 SESSION AND WE WILL BE 3680 02:16:06,019 --> 02:16:08,021 ADDRESSING THIS QUESTIONS DURING 3681 02:16:08,021 --> 02:16:09,055 THE PANEL DISCUSSION. 3682 02:16:09,055 --> 02:16:12,458 OUR FIRST SPEAKER WILL BE 3683 02:16:12,458 --> 02:16:13,960 MR. TRE LAROSA AND HE WILL GIVE 3684 02:16:13,960 --> 02:16:15,728 US A PERSPECTIVE ON CLINICAL 3685 02:16:15,728 --> 02:16:18,831 TRIALS ON PATIENT ENGAINLMENT AS 3686 02:16:18,831 --> 02:16:20,900 AN ADULT WITH CYSTIC FIBROSIS, 3687 02:16:20,900 --> 02:16:23,002 HE ALSO CURRENTLY SERVES ON THE 3688 02:16:23,002 --> 02:16:23,803 CF'S FOUNDATION CLINICAL 3689 02:16:23,803 --> 02:16:26,406 RESEARCH AND EXECUTIVE 3690 02:16:26,406 --> 02:16:27,540 COMMITTEE. 3691 02:16:27,540 --> 02:16:29,475 MR. LAROSA IS ALSO A PROJECT 3692 02:16:29,475 --> 02:16:31,110 MANAGER FOR THE NOGZAL 3693 02:16:31,110 --> 02:16:32,879 FOWBDATION FOR THE NATIONAL 3694 02:16:32,879 --> 02:16:36,049 INSTITUTES OF HEALTH WHERE WHERE 3695 02:16:36,049 --> 02:16:38,685 HE WORKS AS PATIENT AMBASSADOR 3696 02:16:38,685 --> 02:16:41,154 AND WORKS ON METABOLIC 3697 02:16:41,154 --> 02:16:41,421 DISORDERS. 3698 02:16:41,421 --> 02:16:45,191 OUR NEXT SPEAKER WILL BE MICHELE 3699 02:16:45,191 --> 02:16:47,026 MANNION, WHO IS THE EXECUTIVE 3700 02:16:47,026 --> 02:16:48,661 DIRECTOR OF THE PC D FOWBDATION 3701 02:16:48,661 --> 02:16:50,863 WHICH SHE FOUND INDEED 2002. 3702 02:16:50,863 --> 02:16:52,865 SINCE THEN SHE SERVED ON 3703 02:16:52,865 --> 02:16:56,903 NUMEROUS LEADERSHIP ROLE RELATED 3704 02:16:56,903 --> 02:16:58,304 TO POLICY DEVELOPMENT, ACCESS TO 3705 02:16:58,304 --> 02:17:00,406 CARE FOR INDIVIDUALS WITH LUNG 3706 02:17:00,406 --> 02:17:02,008 DISORDERS WITH A HEAVY EMPHASIS 3707 02:17:02,008 --> 02:17:04,811 ON THE PC D COMMUNITY AND SHE'S 3708 02:17:04,811 --> 02:17:05,445 COLLABORATED WITH INVESTIGATORS 3709 02:17:05,445 --> 02:17:06,813 WORLD WIDE AND HAS BEEN A FULL 3710 02:17:06,813 --> 02:17:09,515 PARTNER IN DEVELOPING AND 3711 02:17:09,515 --> 02:17:10,483 SUPPORTING A LARGE PRIMARY 3712 02:17:10,483 --> 02:17:12,318 RESEARCH NETWORK THAT IS IN 3713 02:17:12,318 --> 02:17:13,586 NORTH AMERICA AND EUROPE. 3714 02:17:13,586 --> 02:17:19,492 OUR NEXT SPEAKER WILL BE 3715 02:17:19,492 --> 02:17:20,660 DR. SHERYL NICKERSON NUTTER WHO 3716 02:17:20,660 --> 02:17:22,495 IS THE EXECUTIVE VICE PRESIDENT 3717 02:17:22,495 --> 02:17:24,097 OF RESEARCH AND DEVELOPMENT AT 3718 02:17:24,097 --> 02:17:28,001 THE 3 LAKES FOUNDATION, LEADING 3719 02:17:28,001 --> 02:17:29,135 THERAPIES, PULMONARY FIBROSIS 3720 02:17:29,135 --> 02:17:30,670 AND INTERSTITIAL LUNG DISEASES. 3721 02:17:30,670 --> 02:17:32,171 WITH OR PREVIOUS EXPERIENCE 3722 02:17:32,171 --> 02:17:35,074 SHE'S ADVANCED BOTH BIOLOGIC AND 3723 02:17:35,074 --> 02:17:36,509 SMALL MOLECULE DRUGS INTO 3724 02:17:36,509 --> 02:17:37,910 CLINICAL TRIALS FOR AUTOIMMUNE 3725 02:17:37,910 --> 02:17:38,778 AND INFLAMMATORY DISEASES AND 3726 02:17:38,778 --> 02:17:40,179 SHE CONTINUES TO FOCUS HER 3727 02:17:40,179 --> 02:17:41,981 EFFORTS ON DEVELOPMENT AND MODEL 3728 02:17:41,981 --> 02:17:43,716 SYSTEMS AND BIOMARKERS THAT CAN 3729 02:17:43,716 --> 02:17:44,617 CONTINUE TO SUPPORT CLINICAL 3730 02:17:44,617 --> 02:17:45,151 TRIAL OUTCOMES. 3731 02:17:45,151 --> 02:17:50,890 AND THEN OUR FINAL SPEAKER WILL 3732 02:17:50,890 --> 02:17:54,994 BE DR. FRANCIS Mc CORMACK, WHO 3733 02:17:54,994 --> 02:17:57,997 IS THE DIRECTOR DIVISION OF 3734 02:17:57,997 --> 02:17:59,265 PULMONARY CRITICAL CARE AND 3735 02:17:59,265 --> 02:18:00,433 SCHEEP MEDICINE AT THE 3736 02:18:00,433 --> 02:18:01,701 UNIVERSITY OF SIN SUDDEN ATY 3737 02:18:01,701 --> 02:18:05,772 COLLEGE OF MEDICINE, HE HAS A 3738 02:18:05,772 --> 02:18:09,208 FOCUSED ROLL ON PULMONARY 3739 02:18:09,208 --> 02:18:13,279 IMMUNITY, HIS INTERTITTIAL LUNG 3740 02:18:13,279 --> 02:18:14,647 DISEASES, INCLUDING LAMB, AND 3741 02:18:14,647 --> 02:18:15,548 PRINCIPAL INVESTIGATOR OF THE 3742 02:18:15,548 --> 02:18:18,584 MYLES TRIAL THAT LED TO THE FDA 3743 02:18:18,584 --> 02:18:20,286 APPROVAL OF [INDISCERNIBLE] AS A 3744 02:18:20,286 --> 02:18:22,522 SAFE AND EFFECTIVE TREATMENT 3745 02:18:22,522 --> 02:18:23,356 WITH LAM FOUNDATION, AND WITH 3746 02:18:23,356 --> 02:18:25,224 THAT I WOULD LIKE TO TURN IT 3747 02:18:25,224 --> 02:18:31,698 OVER TO OUR FIRST SPEAKER 3748 02:18:31,698 --> 02:18:33,533 MR. LAROSA. 3749 02:18:33,533 --> 02:18:34,000 >> THANK YOU MARRAH. 3750 02:18:34,000 --> 02:18:35,635 AND FIRST I WANT TO THANK THE 3751 02:18:35,635 --> 02:18:37,203 ORGANIZERS OF THIS WORKSHOP FOR 3752 02:18:37,203 --> 02:18:38,504 INVITING ME TO SPEAK AND PUTTING 3753 02:18:38,504 --> 02:18:39,706 TOGETHER A NICE AGENDA AND 1 3754 02:18:39,706 --> 02:18:41,908 THAT WORKS TO SOLVE THE 3755 02:18:41,908 --> 02:18:43,009 CHALLENGE OF ACTUALIZING 3756 02:18:43,009 --> 02:18:43,476 S TO PATIENTS. 3757 02:18:43,476 --> 02:18:45,411 I ALSO WANT TO DISCLOSE I HAVE 3758 02:18:45,411 --> 02:18:46,879 SERVED IN VARIOUS CAPACITIES FOR 3759 02:18:46,879 --> 02:18:48,481 THE CF FOUNDATION AND THAT MY 3760 02:18:48,481 --> 02:18:52,352 VIEWS ARE MINE AND DO NOT 3761 02:18:52,352 --> 02:18:56,089 REPRESENT MY EMPLOYER, THE FNIH, 3762 02:18:56,089 --> 02:19:01,361 MY TALK WILL WILL BE ABOUT AS AN 3763 02:19:01,361 --> 02:19:05,631 ADULT WITH CYSTIC PHOBEROSIS, 3764 02:19:05,631 --> 02:19:07,800 BUT ALSO AS AN PATIENT 3765 02:19:07,800 --> 02:19:08,768 ENGAGEMENT IN THE PATIENT SPACE. 3766 02:19:08,768 --> 02:19:10,203 CAN YOU PLEASE ADVANCE THE 3767 02:19:10,203 --> 02:19:10,403 SLIDE. 3768 02:19:10,403 --> 02:19:12,472 SO FIRST, A BIT ABOUT ME. 3769 02:19:12,472 --> 02:19:14,607 YOU CAN GO AHEAD AND ADVANCE THE 3770 02:19:14,607 --> 02:19:16,242 SLIDE 1 MORE TIME. 3771 02:19:16,242 --> 02:19:19,379 MY NAME IS TRE LAROSA AND I'M 3772 02:19:19,379 --> 02:19:20,546 KNOWA ADULT WITH 3773 02:19:20,546 --> 02:19:21,247 CYSTIC FIBROSIS, I AM 30 YEARS 3774 02:19:21,247 --> 02:19:25,551 OLD AND I'M A DELTA F508 3775 02:19:25,551 --> 02:19:28,454 HOMOZYGOTE, MEANING I'VE BEEN 3776 02:19:28,454 --> 02:19:35,294 ABLE TO BENEFIT FROM MODALATOR 3777 02:19:35,294 --> 02:19:36,796 THERAPY, TRIKAFTA, AND AS A KID 3778 02:19:36,796 --> 02:19:40,266 MY LIFE WAS FILLED WITH 3779 02:19:40,266 --> 02:19:43,136 APPOINTMENTS, CHEST THERAPY, 3780 02:19:43,136 --> 02:19:46,873 IVs, PILLS, POKES, PRODS, 3781 02:19:46,873 --> 02:19:48,207 SINUS INJURY, COUGH, EXPLAINING 3782 02:19:48,207 --> 02:19:51,244 TO OTHERS MY COULD YOU HAVE WAS 3783 02:19:51,244 --> 02:19:53,579 NOT CONTAGIOUS, INFECTIONS, 3784 02:19:53,579 --> 02:19:54,180 GOOGLE SEARCHES, PULMONARY 3785 02:19:54,180 --> 02:19:55,782 FUNCTION TESTS AND BLOWING SO 3786 02:19:55,782 --> 02:19:58,317 HARD I ENDED UP GETTING 3787 02:19:58,317 --> 02:20:01,521 HEADACHES AND USING GALLOW'S 3788 02:20:01,521 --> 02:20:03,523 [INDISCERNIBLE] THAT COMES WITH 3789 02:20:03,523 --> 02:20:05,658 HAVING A LIFE-SHORTENING ILLNESS 3790 02:20:05,658 --> 02:20:07,593 ISSUES MY EXPERIENCE WHILE IT 3791 02:20:07,593 --> 02:20:09,328 SHARES MANY HALLMARKS OF OF 3792 02:20:09,328 --> 02:20:11,631 OTHERS WITH CF IS BUT 1 3793 02:20:11,631 --> 02:20:12,999 EXPERIENCE. 3794 02:20:12,999 --> 02:20:15,635 IN MY FAMILY'S CASE, IT'S 3795 02:20:15,635 --> 02:20:17,570 GENERATIONAL, MY GRAND PARENTS 3796 02:20:17,570 --> 02:20:19,338 WERE FUNDRAISING IN THE 1960S 3797 02:20:19,338 --> 02:20:20,039 AND SEIVETS. 3798 02:20:20,039 --> 02:20:23,176 MY FATHER LOST HIS SISTER TO 3799 02:20:23,176 --> 02:20:24,677 CYSTIC FIBROSIS TO THIS HORRIFIC 3800 02:20:24,677 --> 02:20:26,112 DEC WHEN SHE WAS ONLY 12 YEARS 3801 02:20:26,112 --> 02:20:28,281 OLD AND HE WAS ONLY 18. 3802 02:20:28,281 --> 02:20:29,615 IT WAS 1968, TREATMENT LOOKEDDA 3803 02:20:29,615 --> 02:20:31,384 I LOT DIFFERENT THEN, THEY 3804 02:20:31,384 --> 02:20:34,120 HADN'T EVEN DISCOVERED THE CFTR 3805 02:20:34,120 --> 02:20:35,188 GENE OR IMPLEMENTED MUCH OF THE 3806 02:20:35,188 --> 02:20:36,956 STANDARD OF CARE WE NOW TAKE FOR 3807 02:20:36,956 --> 02:20:37,290 GRANTED. 3808 02:20:37,290 --> 02:20:39,959 YOU CAN GO AHEAD AND ADVANCE THE 3809 02:20:39,959 --> 02:20:40,259 SLIDE. 3810 02:20:40,259 --> 02:20:41,794 MY EXPERIENCE IS ALSO 1 OF A 3811 02:20:41,794 --> 02:20:43,930 SIBLING OF A LOVED 1 WITH CF. 3812 02:20:43,930 --> 02:20:47,967 IN 1988 MY PARENTS HAD MY SISTER 3813 02:20:47,967 --> 02:20:49,936 ELISSA RENE LAROSA, WHILE WE 3814 02:20:49,936 --> 02:20:51,804 BOTH CARRIED THE MOST COMMON 3815 02:20:51,804 --> 02:20:53,840 MUTATION OF CF OUR EXPERIENCES 3816 02:20:53,840 --> 02:20:55,608 TOED THE OPPOSITE ENDS OF 3817 02:20:55,608 --> 02:20:58,044 PROTOTYPICAL CF AND LOOKED FOR 3818 02:20:58,044 --> 02:20:58,744 DIFFERENT. 3819 02:20:58,744 --> 02:21:00,513 WE BOTH EXHIBITED VERY COMMON CF 3820 02:21:00,513 --> 02:21:02,215 PHENOTYPES BUT I MANAGED TO 3821 02:21:02,215 --> 02:21:04,650 MAINTAIN MY WEIGHT AND WHIEL I 3822 02:21:04,650 --> 02:21:06,519 CULTIVATED COMMON STRAINS OF CF 3823 02:21:06,519 --> 02:21:08,020 BACTERIA, I MANAGED TO BOUNCE 3824 02:21:08,020 --> 02:21:08,855 BACK FROM INFECTIONS QUITE 3825 02:21:08,855 --> 02:21:09,822 QUICKLY MOST OF THE TIME. 3826 02:21:09,822 --> 02:21:13,259 MY SISTER ON THE OTHER HAND 3827 02:21:13,259 --> 02:21:15,127 SPENT 30-40% OF HER LIFE ON 3828 02:21:15,127 --> 02:21:19,098 IVs OR IN THE HOSPITAL. 3829 02:21:19,098 --> 02:21:20,900 EVENTUALLY MY SISTER WOULD 3830 02:21:20,900 --> 02:21:22,502 CULTIVATE A [INDISCERNIBLE] 3831 02:21:22,502 --> 02:21:25,004 VECTOR WHICH TORPIED O'S HER 3832 02:21:25,004 --> 02:21:26,672 FIRST LUNG TRANSPLANT WHEN SHE 3833 02:21:26,672 --> 02:21:29,041 WAS ONLY 25 YEARS OLD. 3834 02:21:29,041 --> 02:21:32,144 SHE DEVELOPED BRONCH YIETIS 3835 02:21:32,144 --> 02:21:37,083 OBLIT RANS, AND NEEDED A 3836 02:21:37,083 --> 02:21:39,952 TRANSPLANT BY 2018, A FEW YEARS 3837 02:21:39,952 --> 02:21:44,557 LATER SHE WOULD DIE. 3838 02:21:44,557 --> 02:21:46,559 I FELT I COULD OVERCOME WHAT CF 3839 02:21:46,559 --> 02:21:48,194 WROTE FOR ME, ON THE OTHER HAND 3840 02:21:48,194 --> 02:21:50,796 CF SHAPED ME IN POSITIVE WAYS, 3841 02:21:50,796 --> 02:21:54,433 TOO, IN FACT, CF COME TO PROVIDE 3842 02:21:54,433 --> 02:21:57,069 ME A CAREER IN THE CF SPACE AND 3843 02:21:57,069 --> 02:21:59,705 NOW MORE BROADLY IN PATIENT AND 3844 02:21:59,705 --> 02:22:00,673 SCIENTIFIC PROJECT MANAGE. 3845 02:22:00,673 --> 02:22:03,075 CF ALSO PROVIDED ME AN INTIMATE 3846 02:22:03,075 --> 02:22:04,243 PERSPECTIVE INTO HUMAN BEHAVIOR. 3847 02:22:04,243 --> 02:22:06,712 FROM A YOUNG AGE I WAS DRAWN TO 3848 02:22:06,712 --> 02:22:08,414 SCIENCE FOR THE POWER TO EXPLAIN 3849 02:22:08,414 --> 02:22:09,815 THE UNKNOWN, SCIENCE IS A TOOL 3850 02:22:09,815 --> 02:22:11,317 WE CAN USE TO UNDERSTAND THE 3851 02:22:11,317 --> 02:22:13,419 WORLD AROUND US AND SCIENCE ALSO 3852 02:22:13,419 --> 02:22:14,387 HELPED US DRAMATICALLY IMPROVE 3853 02:22:14,387 --> 02:22:15,888 OUTCOMES IN THE CF COMMUNITY 3854 02:22:15,888 --> 02:22:19,492 COMPARED TO THE SHORT FATES OF 3855 02:22:19,492 --> 02:22:23,896 PEOPLE SUCH AS MY AUNT AND 3856 02:22:23,896 --> 02:22:25,898 SISTER. 3857 02:22:25,898 --> 02:22:29,068 YOU CAN ADVANCE THE SLIDES. 3858 02:22:29,068 --> 02:22:32,271 THOUGH I DREAMED OF BECOMES A 3859 02:22:32,271 --> 02:22:35,007 DOCTOR, MY CF WOULD CHANGE THOSE 3860 02:22:35,007 --> 02:22:37,543 NEEDS, I WOULD FORGO MEDICINE 3861 02:22:37,543 --> 02:22:38,678 AND WENT INTO SCIENCE. 3862 02:22:38,678 --> 02:22:41,247 I WOULD GO ON TO WORK IN JP 3863 02:22:41,247 --> 02:22:43,115 CLANCY'S CF LAB AT CINCINNATI 3864 02:22:43,115 --> 02:22:43,916 CHILDREN'S MEDICAL CENTER, WHICH 3865 02:22:43,916 --> 02:22:45,585 WAS THE SAME INSTITUTION THAT 3866 02:22:45,585 --> 02:22:47,653 PROVIDED BY CF CARE WHEN I WAS A 3867 02:22:47,653 --> 02:22:48,454 CHILD. 3868 02:22:48,454 --> 02:22:50,156 I ALSO MET DR. [INDISCERNIBLE] 1 3869 02:22:50,156 --> 02:22:52,592 OF THE OTHER SESSION SPEAKERS 3870 02:22:52,592 --> 02:22:56,228 AND DR. [INDISCERNIBLE] 1 OF MY 3871 02:22:56,228 --> 02:22:57,630 CO-PANELISTS WHO TOOK CARE OF MY 3872 02:22:57,630 --> 02:22:58,598 SISTER AT THE UNIVERSITY OF SIN 3873 02:22:58,598 --> 02:22:59,465 SEN ILLEGALSEN ATY. 3874 02:22:59,465 --> 02:23:00,399 AT THIS POINT IN MY LIFE I WOULD 3875 02:23:00,399 --> 02:23:02,401 SPEAK TO I LOT OF CF DOCTORS, 3876 02:23:02,401 --> 02:23:04,270 RESEARCHERS AND OTHER PATIENTS, 3877 02:23:04,270 --> 02:23:05,104 ALL INVOLVED WITH CF RESEARCH 3878 02:23:05,104 --> 02:23:11,010 AND MEDICINE IN DIFFERENT 3879 02:23:11,010 --> 02:23:13,646 CAPACITIES, PEOPLE WHO WERE 3880 02:23:13,646 --> 02:23:15,514 WRITING PROTOCOLS, SHIFT IN 3881 02:23:15,514 --> 02:23:16,482 INCORPORATING PATIENTS TRIAL, 3882 02:23:16,482 --> 02:23:17,416 THE DEVELOPMENT PROCESS, 3883 02:23:17,416 --> 02:23:18,117 PATIENTS PRACTICES RUNNING 3884 02:23:18,117 --> 02:23:20,086 ACROSS THE GAMUT OF PEOPLE 3885 02:23:20,086 --> 02:23:20,720 ELIGIBLE FOR MODULATORS FOCUSED 3886 02:23:20,720 --> 02:23:22,121 ON FOLK WHO IS LIVED IN 3887 02:23:22,121 --> 02:23:23,689 DIFFERENT COUNTRIES AND DIDN'T 3888 02:23:23,689 --> 02:23:26,025 EACH HAVE ACCESS TO STANDARD OF 3889 02:23:26,025 --> 02:23:27,426 CARE SUCH AS HYPERTONIC SALINE. 3890 02:23:27,426 --> 02:23:28,961 I CAME TO REALIZE WE WERE ALL 3891 02:23:28,961 --> 02:23:30,696 TALKING ABOUT THE SAME DETAILS 3892 02:23:30,696 --> 02:23:33,199 IN VERY DIFFERENT LANGUAGE ANDS 3893 02:23:33,199 --> 02:23:34,166 THEREFORE OFTEN TALKING PAST 1 3894 02:23:34,166 --> 02:23:35,768 ANOTHER WHICH INPIERED ME TO 3895 02:23:35,768 --> 02:23:36,902 START WRITING ABOUT MY LIVED 3896 02:23:36,902 --> 02:23:38,137 EXPERIENCE AND THE SCIENCE 3897 02:23:38,137 --> 02:23:39,839 UNDERLYING CF AND BRIDGE THOSE 3898 02:23:39,839 --> 02:23:40,072 GAPS. 3899 02:23:40,072 --> 02:23:41,140 THIS ALSO LED ME TO PATIENT 3900 02:23:41,140 --> 02:23:42,642 ADVOCABULARY KASP A R EXPE 3901 02:23:42,642 --> 02:23:43,242 PARTICIPATE NOTHING CLINICAL 3902 02:23:43,242 --> 02:23:43,509 TRIALS. 3903 02:23:43,509 --> 02:23:47,113 YOU CAN GO AHEAD AND ADVANCE THE 3904 02:23:47,113 --> 02:23:48,080 SLIDE, PLEASE. 3905 02:23:48,080 --> 02:23:49,515 THE FIRST MAJOR CLINICAL TRIAL I 3906 02:23:49,515 --> 02:23:51,250 ENROLLED IN WAS CALLED THE 3907 02:23:51,250 --> 02:23:52,652 IGNITE STUDY WHICH SOUGHT TO 3908 02:23:52,652 --> 02:23:56,355 INVESTIGATE THE USE OF GALLIUM 3909 02:23:56,355 --> 02:23:58,224 NITRATE FOR PSEUDOMOANUS AND CF. 3910 02:23:58,224 --> 02:23:59,458 I WAS FASCINATED BY THIS SINCE I 3911 02:23:59,458 --> 02:24:01,661 WAS WORKING IN A CF LAB, PRIVATE 3912 02:24:01,661 --> 02:24:02,728 SECTOR LODGED ENOUGH TO TAKE 3913 02:24:02,728 --> 02:24:03,996 TIME OFF WORK AND PRIVILEGED 3914 02:24:03,996 --> 02:24:05,965 ENOUGH TO TAKE TIME OFF WORK AND 3915 02:24:05,965 --> 02:24:07,767 LAY IN A HOSPITAL BED AND GET 3916 02:24:07,767 --> 02:24:09,869 PAID TO WATCH SONS OFAN ARCHY 3917 02:24:09,869 --> 02:24:11,904 FOR A WEEK AND WHILE I COULD 3918 02:24:11,904 --> 02:24:12,972 HAVE RECEIVED PLACEBO, I WAS 3919 02:24:12,972 --> 02:24:14,140 EAGER TO SEE WHAT THE STUDY 3920 02:24:14,140 --> 02:24:15,574 WOULD DO FOR MY LUNGS. 3921 02:24:15,574 --> 02:24:18,310 AFTER 5 DAYS MY LUNG FUNCTION 3922 02:24:18,310 --> 02:24:19,745 WENT UP 7%, I RISE I HAD ONLY 3923 02:24:19,745 --> 02:24:21,881 SEEN FROM THE USE OF ANTIBIOTICS 3924 02:24:21,881 --> 02:24:23,282 OR MONTHS OF PHYSICAL TRAINING, 3925 02:24:23,282 --> 02:24:25,151 I THOUGHT THIS BODE WELL FOR THE 3926 02:24:25,151 --> 02:24:26,786 POSSIBILITY OF STUDY MEETING THE 3927 02:24:26,786 --> 02:24:28,187 PRIMARY INFLUENCE AND I 3928 02:24:28,187 --> 02:24:29,388 EVENTUALLY I LEARNED I DID 3929 02:24:29,388 --> 02:24:31,223 RECEIVE DRUG, BUT THE STUDY DID 3930 02:24:31,223 --> 02:24:33,125 NOT REACH PRIMARY END POINTS AND 3931 02:24:33,125 --> 02:24:33,726 I WAS DISAPPOINTED. 3932 02:24:33,726 --> 02:24:35,027 I LEARNED A LOT ABOUT THE 3933 02:24:35,027 --> 02:24:36,862 NUANCES OF STUDY DESIGN, PRIMARY 3934 02:24:36,862 --> 02:24:38,397 POINTS AND HOW IMPORTANT IT WAS 3935 02:24:38,397 --> 02:24:39,765 FORFONTS TO BE INVOLVED IN 3936 02:24:39,765 --> 02:24:41,434 STUDY DESIGN AND HOW IMPORTANT 3937 02:24:41,434 --> 02:24:45,004 IT WAS TO REDUCE BARRIERS TO 3938 02:24:45,004 --> 02:24:46,272 PARTICIPATE NOTHING ADVANCED 3939 02:24:46,272 --> 02:24:46,639 CLINICAL TRIALS. 3940 02:24:46,639 --> 02:24:49,775 PLEASE ADVANCE THE SLIDE. 3941 02:24:49,775 --> 02:24:50,710 I EVENTUALLY JOINED A SMALL 3942 02:24:50,710 --> 02:24:52,611 COMPANY WHERE I WORKED IN 3943 02:24:52,611 --> 02:24:54,714 PATIENT REPORTED OUTCOMES ACROSS 3944 02:24:54,714 --> 02:24:56,816 MANY DISEASES, I ALSO SERVED ON 3945 02:24:56,816 --> 02:24:59,652 MANY COMMITTEES INCLUDING CF 3946 02:24:59,652 --> 02:25:01,387 ADULT ADVISORY COUNCIL, PROTOCOL 3947 02:25:01,387 --> 02:25:03,255 REVIEW COMMITTEE AND 3948 02:25:03,255 --> 02:25:03,856 PATIENT-FACING AND 3949 02:25:03,856 --> 02:25:04,557 SCIENCE-FACING CONFERENCES. 3950 02:25:04,557 --> 02:25:05,524 THESE EXPERIENCES WOULD SHAPE 3951 02:25:05,524 --> 02:25:07,526 THE NEXT MAJOR MILESTONE OF MY 3952 02:25:07,526 --> 02:25:07,727 LIFE. 3953 02:25:07,727 --> 02:25:09,628 JOINING THE CF THERAPEUTIC 3954 02:25:09,628 --> 02:25:12,198 NETWORK DEVELOPMENT CLINICAL 3955 02:25:12,198 --> 02:25:13,232 EXECUTIVE RESEARCH EXECUTIVE 3956 02:25:13,232 --> 02:25:16,736 COMMITTEE KNOWN AS THE CREC, IT 3957 02:25:16,736 --> 02:25:19,438 GOVERNS AND SANCTIONS STUDIES IN 3958 02:25:19,438 --> 02:25:22,074 THE TDM, THIS ROLE WAS 1 OF THE 3959 02:25:22,074 --> 02:25:23,375 MOST MEANINGFUL IN MY LIFE AS IT 3960 02:25:23,375 --> 02:25:25,344 WAS YEARS AND YEARS OF 3961 02:25:25,344 --> 02:25:26,178 CULMINATION IN THE SCIENTIFIC 3962 02:25:26,178 --> 02:25:27,079 SPACE. 3963 02:25:27,079 --> 02:25:28,481 ON THIS COMMITTEE WE ANALYZE THE 3964 02:25:28,481 --> 02:25:31,250 SPECIFIC TRIALS TO THE TDN WHILE 3965 02:25:31,250 --> 02:25:33,319 INSURING WE ARE THE BEST 3966 02:25:33,319 --> 02:25:34,687 POSSIBLE CUSTODIAN FOR THE 3967 02:25:34,687 --> 02:25:37,890 COMMITTEE, WE HAVE TO MINDFUL OF 3968 02:25:37,890 --> 02:25:40,025 THE NUANCES IN THE CF RESEARCH 3969 02:25:40,025 --> 02:25:40,826 AND INCLUDING SAIRCHGZING 3970 02:25:40,826 --> 02:25:42,361 STUDIES THAT DON'T SUCCEED 3971 02:25:42,361 --> 02:25:43,729 EITHER FOR RECRUIT, SAFETY OR 3972 02:25:43,729 --> 02:25:44,864 EFFICACY REASONS. 3973 02:25:44,864 --> 02:25:46,298 I TALK A LOT ABOUT THE 3974 02:25:46,298 --> 02:25:47,500 IMPORTANCE OF HOPE BUT ALSO THE 3975 02:25:47,500 --> 02:25:49,301 PERSON OF SUCCESS, IN MY VIEW, 3976 02:25:49,301 --> 02:25:50,035 PATIENT ENGAGEMENT IN CLINICAL 3977 02:25:50,035 --> 02:25:51,270 RESEARCH IS NOT A EMILY THED 3978 02:25:51,270 --> 02:25:54,340 RESEARCH BUT A FORM OF CAPITAL 3979 02:25:54,340 --> 02:25:55,975 THAT BEGETS ADDITIONAL CAPITAL. 3980 02:25:55,975 --> 02:25:57,376 IN OTHER WORDS PATIENTS THAT 3981 02:25:57,376 --> 02:26:01,647 ENROLL IN CLINICAL TRIALS THAT 3982 02:26:01,647 --> 02:26:03,282 SUCCEED OR LEARN IN THE PROCESS 3983 02:26:03,282 --> 02:26:06,886 ITSELF SUCH AS WHEN I ENROLL 3984 02:26:06,886 --> 02:26:08,053 IN THE IGNITE STUDY. 3985 02:26:08,053 --> 02:26:18,497 YOU CAN ADVANCE THE SLIDE. 3986 02:26:21,467 --> 02:26:22,802 CAN YOU ADVANCE THE SLIDE? 3987 02:26:22,802 --> 02:26:24,937 I THINK THERE SHOULD BE 1 MORE 3988 02:26:24,937 --> 02:26:25,204 SLIDE? 3989 02:26:25,204 --> 02:26:25,471 MAYBE NOT. 3990 02:26:25,471 --> 02:26:27,706 BUT GO AHEAD AND YOU CAN GO BACK 3991 02:26:27,706 --> 02:26:27,840 1. 3992 02:26:27,840 --> 02:26:29,275 TO CONCLUDE MY TALK, I WANT TO 3993 02:26:29,275 --> 02:26:30,342 BRIEFLY TALK ABOUT MY ROLE AT 3994 02:26:30,342 --> 02:26:31,677 THE FOUNDATION FOR THE NATIONAL 3995 02:26:31,677 --> 02:26:32,878 INSTITUTES OF HELT WHICH IS A 3996 02:26:32,878 --> 02:26:33,813 NONPROFIT THAT SEEKS TO ADVANCE 3997 02:26:33,813 --> 02:26:35,581 THE MISSION OF THE NIH. 3998 02:26:35,581 --> 02:26:36,048 HE 3999 02:26:36,048 --> 02:26:38,317 THIS ROLE, WE FACILITATE 4000 02:26:38,317 --> 02:26:39,318 PUBLIC PRIVATE PPUIPS 4001 02:26:39,318 --> 02:26:41,320 BETWEEN THE NIH BEING ACADEMIA 4002 02:26:41,320 --> 02:26:42,621 AND INDUSTRY ORGANIZATIONS. 4003 02:26:42,621 --> 02:26:44,356 I WEAR 2 HATS HERE, I AM A 4004 02:26:44,356 --> 02:26:46,792 SCIENTIFIC PROJECT MANAGER FOR A 4005 02:26:46,792 --> 02:26:47,526 BIOMARKERS CONSORTIUM PROJECT 4006 02:26:47,526 --> 02:26:49,528 SEEKING TO VALENTINED DIADIC A 4007 02:26:49,528 --> 02:26:51,764 PROGNOSTIC BIOMARKER IN 4008 02:26:51,764 --> 02:26:52,932 METABOLIC DYSFUNCTION 4009 02:26:52,932 --> 02:26:53,833 [INDISCERNIBLE] HEPATITIS AND I 4010 02:26:53,833 --> 02:26:55,868 ALSO HELP TO MANAGE PATIENT 4011 02:26:55,868 --> 02:27:00,906 ENGAGEMENTTS. 4012 02:27:00,906 --> 02:27:02,608 THE FNIHTHS A SPECIAL AREA WHERE 4013 02:27:02,608 --> 02:27:05,277 WE GET TO STUDY THINGSA THE A 4014 02:27:05,277 --> 02:27:06,545 DIFFERENT DISEASE PROCESS, EVERY 4015 02:27:06,545 --> 02:27:08,681 AREA IS MATURITY IN TERMS OF 4016 02:27:08,681 --> 02:27:12,051 PATIENT ENGAGEMENT BUT IT'S 4017 02:27:12,051 --> 02:27:13,986 CRUCIAL WHEN CHECKING BOXES FOR 4018 02:27:13,986 --> 02:27:15,754 PEOPLE WITH LIVED EXPERIENCE. 4019 02:27:15,754 --> 02:27:17,456 IT'S NECESSARY TO HELP OPTIMIZE 4020 02:27:17,456 --> 02:27:18,624 DRUG DEVELOPMENT IN THE 4021 02:27:18,624 --> 02:27:19,692 ADVANCEMENT OF THE FIELD FROM 4022 02:27:19,692 --> 02:27:21,460 THE VERY ONSET OF AN IDEA. 4023 02:27:21,460 --> 02:27:23,829 I INVITE THE VOAKS IN THE ROOM 4024 02:27:23,829 --> 02:27:24,663 HERE TODAY TO CHALLENGE 4025 02:27:24,663 --> 02:27:25,598 THEMSELVES TO IMAGINE WHAT IT 4026 02:27:25,598 --> 02:27:28,133 MUST BE LIKE TO BE A PATIENT 4027 02:27:28,133 --> 02:27:29,635 BECAUSE WHILE PATIENTS SHOULD BE 4028 02:27:29,635 --> 02:27:31,237 IN EVERY ROOM, WE AREN'T ALWAYS 4029 02:27:31,237 --> 02:27:32,204 AND THAT'S WHEN IT'S 4030 02:27:32,204 --> 02:27:33,072 PARTICULARLY NECESSARY FOR THE 4031 02:27:33,072 --> 02:27:34,039 DOCTORS AND RESEARCHERS TO 4032 02:27:34,039 --> 02:27:34,506 EXERCISE THAT MUSCLE. 4033 02:27:34,506 --> 02:27:35,941 I LOOK FORWARD TOA TALKING WITH 4034 02:27:35,941 --> 02:27:37,776 YOU MORE AND HAPPY TO MEET TO 4035 02:27:37,776 --> 02:27:38,878 DISCUSS ANYTHING IN GREATER 4036 02:27:38,878 --> 02:27:40,512 DETAIL AND THANK YOU AND I'M 4037 02:27:40,512 --> 02:27:42,448 EXCITED TO PARTICIPATE IN THE 4038 02:27:42,448 --> 02:27:43,549 PANEL LATER. 4039 02:27:43,549 --> 02:27:44,350 >> THANK YOU TRE. 4040 02:27:44,350 --> 02:27:46,218 THAT WAS VERY INSPIRING. 4041 02:27:46,218 --> 02:27:49,054 IN THE PANEL DISCUSSION AND LOOK 4042 02:27:49,054 --> 02:27:52,258 FORWARD TO IT, SO OUR NEXT 4043 02:27:52,258 --> 02:27:53,559 SPEAKER, IS MICHELLE MANNION AND 4044 02:27:53,559 --> 02:28:00,165 SO, I WOULD LIKE I TURN IE L OVR 4045 02:28:00,165 --> 02:28:01,667 TO HER. 4046 02:28:01,667 --> 02:28:02,034 >> HELLO. 4047 02:28:02,034 --> 02:28:06,972 MY NAME IS MICHELE MANNION, AND 4048 02:28:06,972 --> 02:28:08,908 I'M THE EXPECT RETURN OF RESULTS 4049 02:28:08,908 --> 02:28:11,877 DIRECTOR AT PRIMARY CELLULAR 4050 02:28:11,877 --> 02:28:12,511 DISKINNIESIA, IT WAS GREAT TO 4051 02:28:12,511 --> 02:28:13,913 FOLLOW A MEMBER OF THE CF 4052 02:28:13,913 --> 02:28:14,880 FOUNDATION BECAUSE I THINK WHEN 4053 02:28:14,880 --> 02:28:17,549 WE ARE LOOKING AT SUCCESS IN 4054 02:28:17,549 --> 02:28:18,384 THERAPEUTIC DEVELOPMENT FOR A 4055 02:28:18,384 --> 02:28:20,352 RARE DISEASE, THERE IS NO BETTER 4056 02:28:20,352 --> 02:28:24,256 GOLD STANDARD THAN THE 4057 02:28:24,256 --> 02:28:24,823 CYSTIC FIBROSIS FOUNDATION. 4058 02:28:24,823 --> 02:28:26,125 I WANT TO MENTION THAT WHILE I'M 4059 02:28:26,125 --> 02:28:27,793 HERE TO TALK ABOUT PC D AND THE 4060 02:28:27,793 --> 02:28:29,662 EXPERIENCE OF THE PC D 4061 02:28:29,662 --> 02:28:31,897 FOUNDATION, AFTER DOING THIS FOR 4062 02:28:31,897 --> 02:28:34,466 30 YEARS AND HAVING VALUED 4063 02:28:34,466 --> 02:28:36,802 COLLEAGUES ACROSS THE RARE DEC 4064 02:28:36,802 --> 02:28:39,705 SPACE IT HAS BECOME OBVIOUS TO 4065 02:28:39,705 --> 02:28:42,775 ME THAT WHILE WE HAVE A 4066 02:28:42,775 --> 02:28:43,509 DIFFERENT DISEASES WE'RE 4067 02:28:43,509 --> 02:28:45,277 WORKING, BUT A LOT OF THESE 4068 02:28:45,277 --> 02:28:46,445 ADVOCACY GROUPS ARE THE SAME. 4069 02:28:46,445 --> 02:28:48,380 SO CONSIDER OUR EXPERIENCE AS 4070 02:28:48,380 --> 02:28:50,449 REPRESENTATIVE OF THE RARE 4071 02:28:50,449 --> 02:28:52,651 DISEASE EXPERIENCE ESPECIALLY 4072 02:28:52,651 --> 02:28:56,188 FOR LUNG DISEASE. 4073 02:28:56,188 --> 02:28:56,956 NEXT SLIDE. 4074 02:28:56,956 --> 02:29:02,027 SO WHAT IS PC D?IS 4075 02:29:02,027 --> 02:29:07,967 LIKE CYSTIC FIBROSIS, PRIMARY 4076 02:29:07,967 --> 02:29:09,702 SILLIARY DISKINNIESIA, IT'S THE 4077 02:29:09,702 --> 02:29:11,904 DISORDERS OF CLEARANCE, THIS 4078 02:29:11,904 --> 02:29:13,439 ENCLUEDERS SEPARATIVE OF 4079 02:29:13,439 --> 02:29:15,374 AIR WAY DISORDERS, SO ANY AIR 4080 02:29:15,374 --> 02:29:17,042 WAY INFECTIOUS KIND OF DISEASE 4081 02:29:17,042 --> 02:29:18,410 WOULD BE CATEGORIZED IN THIS 4082 02:29:18,410 --> 02:29:21,480 AREA AND WE'RE TALKING HERE 4083 02:29:21,480 --> 02:29:22,247 ABOUT GENETIC DISORDERS. 4084 02:29:22,247 --> 02:29:25,918 THE ESTISS THAT THERE ARE 4085 02:29:25,918 --> 02:29:27,886 ABOUT 1 IN 7500 TO 1 IN 15,000 4086 02:29:27,886 --> 02:29:30,723 PEOPLE WITH PC D, IT AFFECTS ALL 4087 02:29:30,723 --> 02:29:34,927 GENDERS,LLS, ETHNICITIES, RACESD 4088 02:29:34,927 --> 02:29:38,731 DESPITE THE FACT THAT ORGAN 4089 02:29:38,731 --> 02:29:39,765 PLACEMENT ABNORMALITIES, THESE 4090 02:29:39,765 --> 02:29:41,400 EFFECTS OCCUR IN 50% OF THE 4091 02:29:41,400 --> 02:29:42,601 POPULATION, DIAGNOSIS AND STILL 4092 02:29:42,601 --> 02:29:44,803 OFTEN DELAYED BY YEARS OR EVEN 4093 02:29:44,803 --> 02:29:47,373 DECADES, THERE ARE CURRENTLY NO 4094 02:29:47,373 --> 02:29:48,474 APPROVED THERAPIES FOR PC D. 4095 02:29:48,474 --> 02:29:50,676 SO THE COMMON SYMPTOMS OF PC D, 4096 02:29:50,676 --> 02:29:52,678 I MENTIONED THE LATERALITY 4097 02:29:52,678 --> 02:29:55,848 DEFECTS BUT RIGHT AWAY, THE 4098 02:29:55,848 --> 02:29:56,782 SYMPTOMS ARE EVIDENT AT BIRTH. 4099 02:29:56,782 --> 02:29:58,317 MOST OF THE PATIENT POPULATION, 4100 02:29:58,317 --> 02:30:02,721 MORE THAN 80% WILL HAVE NEONATAL 4101 02:30:02,721 --> 02:30:03,288 RESPIRATTRESS DESPITE 4102 02:30:03,288 --> 02:30:05,324 BIRTH ANDEN THIS MOSTLY, 4103 02:30:05,324 --> 02:30:08,160 THAT IS A SURVIVABLE CONDITION 4104 02:30:08,160 --> 02:30:10,029 ALTHOUGH SOME BABIES DO NOT 4105 02:30:10,029 --> 02:30:11,563 SURVIVE THAT NEONATAL PERIOD BUT 4106 02:30:11,563 --> 02:30:12,631 AS THEY GROW, FIRST YEAR OF 4107 02:30:12,631 --> 02:30:14,733 LIFE, WE START WITH CHRONIC 4108 02:30:14,733 --> 02:30:17,236 UPPER AND LOWER AIR WAY 4109 02:30:17,236 --> 02:30:17,536 INFECTIONS. 4110 02:30:17,536 --> 02:30:19,738 HEARING LOSS IS VERY COMMON IN 4111 02:30:19,738 --> 02:30:23,442 CHILDREN, BOTHIVE AND 4112 02:30:23,442 --> 02:30:25,444 NEUROGENIC, AS PATIENTS PROGRESS 4113 02:30:25,444 --> 02:30:26,478 THEY GET INCREASINGLY BAD BUGS 4114 02:30:26,478 --> 02:30:27,880 AND AIR WAY INFECTIONS LEADING 4115 02:30:27,880 --> 02:30:30,883 TO DAMAGE TO THE AIR WAYS CALL 4116 02:30:30,883 --> 02:30:33,018 BRONCHIECTASIS AND THIS LEADS 4117 02:30:33,018 --> 02:30:35,454 THEM TO TRANSPLANT OR EARLY 4118 02:30:35,454 --> 02:30:38,624 RESPIRATORY FAILURE. 4119 02:30:38,624 --> 02:30:39,658 ADDITIONALLY, SILLIARY ARE 4120 02:30:39,658 --> 02:30:41,660 IMPORTANT FOR ORGANS OF 4121 02:30:41,660 --> 02:30:42,361 PRODUCTION, SO INFERTILITY ARE 4122 02:30:42,361 --> 02:30:44,763 SEEN IN THESE PATIENTS. 4123 02:30:44,763 --> 02:30:47,800 LESS COMMONLY PC D CAN BE 4124 02:30:47,800 --> 02:30:49,234 ASSOCIATE WIDE SYNDROMIC FORMS 4125 02:30:49,234 --> 02:30:51,970 OR X-LINKED FORMS AND YOU WILL 4126 02:30:51,970 --> 02:30:58,477 SEE THINGS LIKE RETINAL 4127 02:30:58,477 --> 02:31:00,579 LOCATIONINNITEIS PIGMENTOSA, AND 4128 02:31:00,579 --> 02:31:01,447 BRAIN MALFORMATIONS, AND 4129 02:31:01,447 --> 02:31:02,581 OCCASIONALLY DEFEBTS THAT ARE 4130 02:31:02,581 --> 02:31:04,516 ASSOCIATE WIDE LATERALITY ISSUES 4131 02:31:04,516 --> 02:31:09,922 CAN BE QUITE SEVERE INCLUEDINGRT 4132 02:31:09,922 --> 02:31:12,224 DEFECS, CHILDREN WITH PC D WITH 4133 02:31:12,224 --> 02:31:13,992 SITUS AMBIGUOUS IS WHAT WE CALL 4134 02:31:13,992 --> 02:31:15,027 THIS CATEGORY. 4135 02:31:15,027 --> 02:31:19,231 OF HAVING CONGENITAL HEARTRISK 4136 02:31:19,231 --> 02:31:22,401 DEFECTS AND WELL IS A 4137 02:31:22,401 --> 02:31:25,204 INCREASE IN SKELETAL ANOMALIES 4138 02:31:25,204 --> 02:31:26,438 INCLUDING SCOLIOSIS AND 4139 02:31:26,438 --> 02:31:29,274 [INDISCERNIBLE] IN THIS PATIENT 4140 02:31:29,274 --> 02:31:29,741 GROUP. 4141 02:31:29,741 --> 02:31:30,042 NEXT SLIDE. 4142 02:31:30,042 --> 02:31:33,912 SO WHAT ARE CILIA? 4143 02:31:33,912 --> 02:31:35,747 WHAT WE'RE TALKING ABOUT WITH PC 4144 02:31:35,747 --> 02:31:39,084 D ARE MODAL CILIA, THERE ARE 4145 02:31:39,084 --> 02:31:40,052 SINGLE PRIMARY/SENSORY 4146 02:31:40,052 --> 02:31:41,587 SILLIARYIA, IN EVERYIAELL OF THE 4147 02:31:41,587 --> 02:31:42,754 BODY E RED BLOOD CELLS BUT 4148 02:31:42,754 --> 02:31:44,189 THEY'RE NOT WHAEY TALKING 4149 02:31:44,189 --> 02:31:47,059 ABOUT WITH PC D, IT IS ONLY THE 4150 02:31:47,059 --> 02:31:48,694 CATEGORY OF MODAL CILIA, AND YOU 4151 02:31:48,694 --> 02:31:52,097 CAN SEE HERE THAT THEY KIND OF 4152 02:31:52,097 --> 02:31:55,067 GROUP TOGETHER IN THE CILIATED 4153 02:31:55,067 --> 02:31:56,135 EPITHELIUM AND THE ROLE IS TO 4154 02:31:56,135 --> 02:31:59,505 MOVE BACK AND FORTH IN A 4155 02:31:59,505 --> 02:32:03,509 SWEEPING FASHION TO GET MUCUS 4156 02:32:03,509 --> 02:32:06,778 THE AIROHIS IS ADEBRIS OUT O 4157 02:32:06,778 --> 02:32:08,647 CRITICAL PART OF HOST, PULMONARY 4158 02:32:08,647 --> 02:32:13,418 HOST DEFENSE AND IN DISORDERS 4159 02:32:13,418 --> 02:32:14,520 UPON OF GENETIC, IT IS 4160 02:32:14,520 --> 02:32:14,786 EFFECTIVE. 4161 02:32:14,786 --> 02:32:17,089 IN THE CF IS THE MUCUS THAT IS 4162 02:32:17,089 --> 02:32:18,857 THE PRIMARY PROBLEM, IN PC D IT 4163 02:32:18,857 --> 02:32:21,760 IS THE CILIA. 4164 02:32:21,760 --> 02:32:22,327 NEXT SLIDE. 4165 02:32:22,327 --> 02:32:24,930 SO WHEN YOU SEE PICTURES OF 4166 02:32:24,930 --> 02:32:26,665 CILIA, THEY LOOK PRETTY SIMPLE, 4167 02:32:26,665 --> 02:32:28,200 THEY LOOK LIKE LITTLE HAIR OR 4168 02:32:28,200 --> 02:32:29,701 WHIP LIKE STRUCTURES BUT THEY 4169 02:32:29,701 --> 02:32:31,403 ACTUALLY ARE VERY COMPLEX. 4170 02:32:31,403 --> 02:32:35,174 SO THIS IS A GRAPHIC OF A MODAL 4171 02:32:35,174 --> 02:32:37,276 CILIUM AND YOU CAN SEE THAT IT'S 4172 02:32:37,276 --> 02:32:38,477 ANCHORED TO A BASE AND THIS IS 4173 02:32:38,477 --> 02:32:40,879 WHAT IS ON THE EPITHELIAL 4174 02:32:40,879 --> 02:32:45,651 THAT ENTIRE STRUCTURE 4175 02:32:45,651 --> 02:32:47,286 WITH THE SILLIARY AXINE ANDIL TE 4176 02:32:47,286 --> 02:32:48,987 BASE IS OVER 500 PROTEINS, 5 SOF 4177 02:32:48,987 --> 02:32:51,089 WE LOOK JUST AT THE PART THAT IS 4178 02:32:51,089 --> 02:32:54,993 INVOLVED IN PC D, AXONINE 4179 02:32:54,993 --> 02:32:57,062 AND DO A CROSS SECTION, CAN YOU 4180 02:32:57,062 --> 02:32:58,664 SEE ALL THESE DIFFERENT 4181 02:32:58,664 --> 02:32:59,097 STRUCTURAL ELEMENTS. 4182 02:32:59,097 --> 02:33:00,299 ALL OF THEM HAVE A PROTEIN 4183 02:33:00,299 --> 02:33:01,366 INVOLVE WIDE THEM AND THE 4184 02:33:01,366 --> 02:33:04,036 ESTIMATE IS THAT THERE ARE ABOUT 4185 02:33:04,036 --> 02:33:06,905 200 SEPARATE PROTEINS INVOLVED 4186 02:33:06,905 --> 02:33:11,510 IN THESE -- CREATING THESE ARMS 4187 02:33:11,510 --> 02:33:15,714 ORSEHEXONINES. 4188 02:33:15,714 --> 02:33:16,848 NEXT SLIDE. 4189 02:33:16,848 --> 02:33:18,951 SO WHEN CILIA DON'T WORK THERE 4190 02:33:18,951 --> 02:33:20,452 ARE MULTIPLE CLINICAL 4191 02:33:20,452 --> 02:33:21,153 CONSEQUENCES. 4192 02:33:21,153 --> 02:33:22,888 WE'VE TALKED A LITTLE BIT ABOUT 4193 02:33:22,888 --> 02:33:25,524 THE ORGAN PLACEMENT AND 4194 02:33:25,524 --> 02:33:28,427 LATERALITY DEFECTS AND THE 4195 02:33:28,427 --> 02:33:29,461 ASSOCIATION WITH CONGENITAL 4196 02:33:29,461 --> 02:33:32,297 HEART DEFECTS, THERE ARE ALSO 4197 02:33:32,297 --> 02:33:34,700 FOUND IN THE APPEND MALCELLS OF 4198 02:33:34,700 --> 02:33:36,001 THE BRAIN, THE CELLS OF THE 4199 02:33:36,001 --> 02:33:39,204 BRAIN SO SOMETIMES YOU WILL SEE 4200 02:33:39,204 --> 02:33:41,740 HYDROCEPHALUS IN PC D, EAR 4201 02:33:41,740 --> 02:33:44,209 INFECTED BY THE PC D, UPPER AND 4202 02:33:44,209 --> 02:33:45,277 LOWER RESPIRATORY TRACT ARE 4203 02:33:45,277 --> 02:33:47,246 PROBABLY THE PRIMARY AREA OF 4204 02:33:47,246 --> 02:33:49,481 MORBIDITY IN PC D, BUT THERE ARE 4205 02:33:49,481 --> 02:33:53,585 ALSO ISSUES WITH REPRODUCTION 4206 02:33:53,585 --> 02:33:58,023 BECAUSE OF CILIA IN THE OVERDUCT 4207 02:33:58,023 --> 02:33:59,324 AND BECAUSE SPERM FLAGELLA HAVE 4208 02:33:59,324 --> 02:34:02,127 THE SAME MOTOR PROTEINS THAT 4209 02:34:02,127 --> 02:34:02,995 AIRWAY CILIA HAVE. 4210 02:34:02,995 --> 02:34:04,863 SO THE GRAPHIC TO THE RIGHT, 4211 02:34:04,863 --> 02:34:08,100 JUST SHOWS THE KIND OF 4 BUCKETS 4212 02:34:08,100 --> 02:34:16,241 OF LATERALITY THAT WE SEE IN PC 4213 02:34:16,241 --> 02:34:18,477 D, SITUS, SOLITUS, THAT'S THE 4214 02:34:18,477 --> 02:34:23,649 TYPICAL, AND THE SITUS, 4215 02:34:23,649 --> 02:34:26,852 INVERSUS, TOTALIS, WHICH IS 4216 02:34:26,852 --> 02:34:29,488 REVERSED, AND THEN SITUS, 4217 02:34:29,488 --> 02:34:30,989 AMBIGUOUS OR HETEROGENEOUS ORY 4218 02:34:30,989 --> 02:34:33,292 TAXI WHICH CAN HAVE 4219 02:34:33,292 --> 02:34:34,926 DEVELOPMENTAL 4220 02:34:34,926 --> 02:34:39,965 ENDOCRINOLOGYINATING EFFECTS. 4221 02:34:39,965 --> 02:34:40,632 NEXT SLIDE. 4222 02:34:40,632 --> 02:34:44,269 SO I GOT INTO THIS FOR THE SAME 4223 02:34:44,269 --> 02:34:48,040 REASON MOST PEOPLERE DO TO 4224 02:34:48,040 --> 02:34:49,174 ADVOCATE FOR THIS DISEASE, AT 4225 02:34:49,174 --> 02:34:52,944 THE TIME WHEN SHE WAS DIAGNOSED 4226 02:34:52,944 --> 02:34:54,579 IN 2001 WAS WHERE CAN WE TAKE 4227 02:34:54,579 --> 02:34:57,382 HER TO GET RESEARCH AND ANSWERS, 4228 02:34:57,382 --> 02:35:00,085 I WAS TOLD THERE WOULD NEVER BE 4229 02:35:00,085 --> 02:35:03,722 RESEARCH ON THIS DISEASE, IT WAS 4230 02:35:03,722 --> 02:35:05,891 TOO RARE AND NOT APPEALINGDO 4231 02:35:05,891 --> 02:35:06,158 RESEARCH. 4232 02:35:06,158 --> 02:35:07,359 AT THIS TIME THERE WAS 4233 02:35:07,359 --> 02:35:08,593 , SO I WHAT LITTLE 4234 02:35:08,593 --> 02:35:09,828 RESEARCH ITLOULDSE AT THE LIBRAY 4235 02:35:09,828 --> 02:35:15,300 TRYING TO FIND ANY SORT OF AOR 4236 02:35:15,300 --> 02:35:16,101 ORGANIZATION FOR THIS DISORDER 4237 02:35:16,101 --> 02:35:19,037 AND THERE WAS NOTHING. 4238 02:35:19,037 --> 02:35:21,473 SO AS THE EARLY INTERNET CAME 4239 02:35:21,473 --> 02:35:22,974 ONLINE, I CHECKED EVERY NOW AND 4240 02:35:22,974 --> 02:35:25,510 THEN AND FINALLY IN 1991 FOUND 4241 02:35:25,510 --> 02:35:26,778 THERE WAS RESEARCH GOING ON AT 4242 02:35:26,778 --> 02:35:28,914 THE UNIVERSITY OF NORTH CAROLINA 4243 02:35:28,914 --> 02:35:29,514 CHAPEL HILL. 4244 02:35:29,514 --> 02:35:31,783 AND TOOK MY DAUGHTER DOWN THEREW 4245 02:35:31,783 --> 02:35:33,852 T THAT WAS T REALLY THEEA TS 4246 02:35:33,852 --> 02:35:36,722 OF THINKING MAYBE WE COULD DO 4247 02:35:36,722 --> 02:35:38,256 SOMETHING HERE. 4248 02:35:38,256 --> 02:35:39,858 AND WE HAD BECAUSE OF THE CLOSE 4249 02:35:39,858 --> 02:35:40,492 ASSOCIATION WITH 4250 02:35:40,492 --> 02:35:44,463 CYSTIC FIBROSIS, WE HAD A LOT OF 4251 02:35:44,463 --> 02:35:45,097 SIMILAR DOCTORS. 4252 02:35:45,097 --> 02:35:46,631 WE ARE SHARING PHYSICIANS, WE 4253 02:35:46,631 --> 02:35:48,700 HAD A LOT OF SIMILAR TREATMENT 4254 02:35:48,700 --> 02:35:50,769 NEEDS AND SO IT MADE SENSE TO 4255 02:35:50,769 --> 02:35:53,071 FOLLOW WHAT THE CF FOUNDATION 4256 02:35:53,071 --> 02:35:55,540 HAD DONE FOR PC D ONLY DOING IT 4257 02:35:55,540 --> 02:35:58,310 WITH MUCH, MUCED BUDGET. 4258 02:35:58,310 --> 02:36:00,045 BUT OUR MISSION FROM THE 4259 02:36:00,045 --> 02:36:01,446 BEGINNING WAS TO BE GOOD 4260 02:36:01,446 --> 02:36:03,148 PARTNERS TO MAKE SURE RESEARCH 4261 02:36:03,148 --> 02:36:04,549 COULD HAPPEN BECAUSE OUR FEELING 4262 02:36:04,549 --> 02:36:06,218 WAS THE BEST WAY TO SUPPORT 4263 02:36:06,218 --> 02:36:08,220 PATIENTS IS TO MAKE SURE THEY 4264 02:36:08,220 --> 02:36:10,322 HAVE ACCESS TO THE BEST CARE IS 4265 02:36:10,322 --> 02:36:12,023 THE THERAPIES THEY NEED. 4266 02:36:12,023 --> 02:36:14,459 SO WE ESTABLISHED OURSELVES AS A 4267 02:36:14,459 --> 02:36:16,695 NONPROFIT ORGANIZATION IN 2001 4268 02:36:16,695 --> 02:36:20,899 AND BECAME TAX EXEMPT, AND THEN 4269 02:36:20,899 --> 02:36:22,167 SERENDIPITOUSLY IN 2002, THE 4270 02:36:22,167 --> 02:36:25,337 RARE DECS CLINICAL RESEARCH 4271 02:36:25,337 --> 02:36:27,072 NETWORK WAS FORMED AND WE BECAME 4272 02:36:27,072 --> 02:36:29,775 1 OF THE FIRST RESEARCH 4273 02:36:29,775 --> 02:36:32,310 CONSORTIA THROUGH THAT PROGRAM 4274 02:36:32,310 --> 02:36:38,216 WITH THE GENETIC DISEASE, 4275 02:36:38,216 --> 02:36:39,651 DISORDERS AND MUCUS SILLARY 4276 02:36:39,651 --> 02:36:41,253 CONSORTIUM AND THAT START INDEED 4277 02:36:41,253 --> 02:36:41,453 2002. 4278 02:36:41,453 --> 02:36:43,121 ONE THING I WANT TO MENTION 4279 02:36:43,121 --> 02:36:44,322 BECAUSE IT'S RELEVANT FOR A LOT 4280 02:36:44,322 --> 02:36:47,292 OF PATIENT GROUPS IS THAT WE ARE 4281 02:36:47,292 --> 02:36:50,429 ASKED TO DO A LOT AS PATIENT 4282 02:36:50,429 --> 02:36:52,731 GROUPS AND MOST GROUPS, THIS 4283 02:36:52,731 --> 02:36:54,599 IS A VOLUNTEER EFFORT AND WE'VE 4284 02:36:54,599 --> 02:36:56,168 BEEN TALKING ABOUTEN THIS A LOT 4285 02:36:56,168 --> 02:36:57,702 INTERNATIONALLY WITH SOME OF THE 4286 02:36:57,702 --> 02:36:59,204 PATIENT ADVOCACY GROUPS FOR PC D 4287 02:36:59,204 --> 02:37:01,306 AND OTHER PARTS OF THE WORLD. 4288 02:37:01,306 --> 02:37:02,941 THINGS REALLY CHANGE FOR US, AND 4289 02:37:02,941 --> 02:37:04,009 MOVED MUCH MORE QUICKLY WHEN I 4290 02:37:04,009 --> 02:37:06,378 WAS ABLE TO DO THIS ASS A 4291 02:37:06,378 --> 02:37:09,014 WHEN WE HAD THE RESOURCES TO 4292 02:37:09,014 --> 02:37:11,216 REALLY INVEST IN THE 4293 02:37:11,216 --> 02:37:12,117 ORGANIZATION, AND SEVERAL YEARS 4294 02:37:12,117 --> 02:37:15,220 AGO I WAS CALLED BY SOMEBODY 4295 02:37:15,220 --> 02:37:17,055 FROM NCATS WHO ASKED ME IF I HAD 4296 02:37:17,055 --> 02:37:20,192 A SENSE FOR HOW MANY RARE DEC 4297 02:37:20,192 --> 02:37:22,060 ORGA HADGA A 4298 02:37:22,060 --> 02:37:23,895 REVENUE OFON OR MORE AND 4299 02:37:23,895 --> 02:37:27,132 WHEN I GOT DONE LAUGHING, I SAID 4300 02:37:27,132 --> 02:37:28,333 POSSIBLE LE 2-3% BUT THAT WOULD 4301 02:37:28,333 --> 02:37:29,267 BE ABOUT IT. 4302 02:37:29,267 --> 02:37:31,002 THE CF FOUNDATION IS A UNICORN 4303 02:37:31,002 --> 02:37:33,505 IN THIS SPACE, MOST OF US ARE 4304 02:37:33,505 --> 02:37:34,840 OPERATING ON VERY, VERY LIMITED 4305 02:37:34,840 --> 02:37:36,208 RESOURCES L WE THINK THAT'S 4306 02:37:36,208 --> 02:37:38,376 IMPORTANT TO MAKE CLEAR TO 4307 02:37:38,376 --> 02:37:44,115 POTENTIALNT PARTNERS. 4308 02:37:44,115 --> 02:37:44,883 NEXT SLIDE. 4309 02:37:44,883 --> 02:37:47,352 SO WHEN THE GDMCC WAS FORMED 4310 02:37:47,352 --> 02:37:48,053 THIS WAS AN ITHE 4311 02:37:48,053 --> 02:37:50,555 OPPORTUNITY FOR US TO GET 4312 02:37:50,555 --> 02:37:51,756 FOUNDATIONAL RESEARCH DONE IN PC 4313 02:37:51,756 --> 02:37:55,894 D THAT WE WOULD NOT HAVE HAD THE 4314 02:37:55,894 --> 02:37:58,096 ABILITY TO PAY FOR PROBABLY TO 4315 02:37:58,096 --> 02:38:00,765 THIS DAY, SO I THINK LOOKING FOR 4316 02:38:00,765 --> 02:38:02,267 OPPORTUNITIES LIKE THIS, WHERE 4317 02:38:02,267 --> 02:38:05,136 YOU CAN HAVE COLLABORATIVE 4318 02:38:05,136 --> 02:38:06,505 RESEARCH CONSORTIA, ORRE CAN 4319 02:38:06,505 --> 02:38:08,707 LOOK FOR OPPORTUNITIES 4320 02:38:08,707 --> 02:38:09,774 THROUGH PRIVATE VENTURES, IT'S 4321 02:38:09,774 --> 02:38:12,444 REALLY IMPORTANT TO GET THAT 4322 02:38:12,444 --> 02:38:13,745 KIND OF BOOST TO MOVE THINGS 4323 02:38:13,745 --> 02:38:16,648 ALONG AND WHAT HAPPENED THROUGH 4324 02:38:16,648 --> 02:38:19,651 THE GDMCC IS THAT WE STARTED 4325 02:38:19,651 --> 02:38:22,120 SMALL WITH 5 SITES AND THEN WE 4326 02:38:22,120 --> 02:38:23,655 GREW OVER TIME AND WHAT BECAME 4327 02:38:23,655 --> 02:38:25,624 CLEAR AT LEAST TO ME AS AN 4328 02:38:25,624 --> 02:38:26,858 OBSERVER AND PARTNER IN THIS 4329 02:38:26,858 --> 02:38:28,627 PROGRAM, IS THAT YEAH, THERE IS 4330 02:38:28,627 --> 02:38:32,564 INTEREST AND IF WE BUILD A 4331 02:38:32,564 --> 02:38:37,202 NETWORK, WE WILL FIND PATIENTS. 4332 02:38:37,202 --> 02:38:37,502 NEXT SLIDE. 4333 02:38:37,502 --> 02:38:41,373 ANDNE SO FROM THAT, THE PC D 4334 02:38:41,373 --> 02:38:42,841 FOUNDATION, PATH TO CLINICAL 4335 02:38:42,841 --> 02:38:45,243 TRIALS WAS ESTABLISHED AND THIS 4336 02:38:45,243 --> 02:38:50,782 REALLY WAS AN EFFORT ON OUR OPAC 4337 02:38:50,782 --> 02:38:52,317 HERE FROM THE CF FOUNDATION, WE 4338 02:38:52,317 --> 02:38:54,519 CALLED HIM AND SAID BOB WE WANT 4339 02:38:54,519 --> 02:38:56,655 TO DO WHAT YOU GUYS DID, WE WANT 4340 02:38:56,655 --> 02:38:58,056 A CENTERS NETWORK AND WE WANT A 4341 02:38:58,056 --> 02:39:00,091 REGISTRY AND OH BY THE WAY WE 4342 02:39:00,091 --> 02:39:00,992 HAVE ABOUT $2500. 4343 02:39:00,992 --> 02:39:03,128 AND TO HIS CREDIT HE DIDN'T 4344 02:39:03,128 --> 02:39:04,996 LAUGH OR HANG UP ON ME. 4345 02:39:04,996 --> 02:39:07,198 BUT HE SAID, YOU KNOW WHAT I 4346 02:39:07,198 --> 02:39:09,601 WOULD DO IS PUT THIS PACKAGE IT 4347 02:39:09,601 --> 02:39:10,769 INTO SOMETHING CALLED THE PATH 4348 02:39:10,769 --> 02:39:12,137 TO CLINICAL TRIALS AND TRY TO 4349 02:39:12,137 --> 02:39:12,671 RAISE MONEY. 4350 02:39:12,671 --> 02:39:14,940 SO THAT IS EXACTLY WHAT WE DID 4351 02:39:14,940 --> 02:39:19,511 AS AN OUTGROWTH OF THE GDMCC. 4352 02:39:19,511 --> 02:39:21,613 THE PC D FOUNDATION CREATED A 4353 02:39:21,613 --> 02:39:25,016 CENTERS NETWORK AND LAUNCHED A 4354 02:39:25,016 --> 02:39:25,283 REGISTRY. 4355 02:39:25,283 --> 02:39:25,784 NEXT SLIDE. 4356 02:39:25,784 --> 02:39:28,453 AND SO HERE ARE SITES TODAY, WE 4357 02:39:28,453 --> 02:39:33,491 HAVE 63 SITES INCLUDING THE 8 4358 02:39:33,491 --> 02:39:35,060 GDMCC SITES, THESE SITES ARE ALL 4359 02:39:35,060 --> 02:39:36,895 ACCREDITED AND HAVE AGREED TO DO 4360 02:39:36,895 --> 02:39:38,630 -- FOLLOW THE GUIDELINES FOR 4361 02:39:38,630 --> 02:39:40,365 DIAGNOSIS AND TO PARTICIPATE IN 4362 02:39:40,365 --> 02:39:41,766 OUR RESEARCH REGISTRY AND ALSO 4363 02:39:41,766 --> 02:39:42,834 TO PARTICIPATE IN RESEARCH 4364 02:39:42,834 --> 02:39:48,206 OPPORTUNITIES AS THEY COME UP. 4365 02:39:48,206 --> 02:39:49,074 NEXT SLIDE. 4366 02:39:49,074 --> 02:39:51,409 AND SO OUR REGISTRY STARTED 4367 02:39:51,409 --> 02:39:53,311 ABOUT 4 YEARS AGO, STILL PRETTY 4368 02:39:53,311 --> 02:39:55,380 SMALL, BUT WE ARE BEING VERY 4369 02:39:55,380 --> 02:39:57,015 CAUTIOUS ABOUT WHO WE PUT IN THE 4370 02:39:57,015 --> 02:39:58,617 REGISTRY BECAUSE WE HAVE A BIG 4371 02:39:58,617 --> 02:40:00,518 PROBLEM, NOT JUST WITH MISSED 4372 02:40:00,518 --> 02:40:02,387 DIAGNOSIS, IN PC D BUT WITH 4373 02:40:02,387 --> 02:40:04,456 MISDIAGNOSIS, SO ALL OF THE 4374 02:40:04,456 --> 02:40:05,523 PATIENTS IN OUR REGISTRY, WE 4375 02:40:05,523 --> 02:40:08,126 WANT THEM TO BE RESEARCH READY, 4376 02:40:08,126 --> 02:40:11,496 MEANING THE DIAGNOSIS HAS TO BE 4377 02:40:11,496 --> 02:40:13,865 CONFIRMED GENETICALLY, OR BY 4378 02:40:13,865 --> 02:40:15,133 HALLMARK THE STRUCTURAL 4379 02:40:15,133 --> 02:40:17,535 DEFECS THAT ARE KNOWN TO CAUSE 4380 02:40:17,535 --> 02:40:18,136 PC D. 4381 02:40:18,136 --> 02:40:19,537 I WOULD LIKE TO POINT OUT HERE 4382 02:40:19,537 --> 02:40:20,739 THAT WE'RE TRYING VERY HARD TO 4383 02:40:20,739 --> 02:40:22,340 GROW OUR ADULT SIDE OF THIS AND 4384 02:40:22,340 --> 02:40:27,512 PATIENTS IN OUR REGISTRY RIGHT 4385 02:40:27,512 --> 02:40:30,782 NOW HAVE CONFIRMED PC D VIA 4386 02:40:30,782 --> 02:40:31,416 GENETICS. 4387 02:40:31,416 --> 02:40:31,716 NEXT SLIDE. 4388 02:40:31,716 --> 02:40:36,354 WE ALSO ARE TRYING TO FOLLOW 4389 02:40:36,354 --> 02:40:38,857 CF FOUNDATION'S MODEL IN HAVING 4390 02:40:38,857 --> 02:40:40,258 A DRUG DEVELOPMENT PIPELINE. 4391 02:40:40,258 --> 02:40:42,193 SO THIS IS A LITTLE BIT 4392 02:40:42,193 --> 02:40:43,928 AMBITIOUS, BECAUSE THERE ARE NOT 4393 02:40:43,928 --> 02:40:48,366 REALLY MANY DRUGS DEVELOPED -- 4394 02:40:48,366 --> 02:40:50,035 IN DEVELOPMENT FOR PC D, AND 4395 02:40:50,035 --> 02:40:53,038 NONE APPROVED FOR PC D.CT IF THN 4396 02:40:53,038 --> 02:40:54,239 DEVELOPMENT, IF THERE ARE DRUGS 4397 02:40:54,239 --> 02:40:57,108 WE THINK WOULD BE APPROPRIATE 4398 02:40:57,108 --> 02:40:58,476 FOR OUR POPULATION, WE PUT THEM 4399 02:40:58,476 --> 02:41:00,445 ON THIS PIPELINE BECAUSE WE B WT 4400 02:41:00,445 --> 02:41:02,447 TS TO KNOW THAT THERE'S 4401 02:41:02,447 --> 02:41:04,883 HELP COMING ANDOMHAT WE WERE 4402 02:41:04,883 --> 02:41:11,823 WORKING THIS. 4403 02:41:11,823 --> 02:41:12,424 NEXTLIDE. 4404 02:41:12,424 --> 02:41:17,128 SO WE'VE DONE A COUPLE CLINICAL 4405 02:41:17,128 --> 02:41:18,430 TRIALS IN PC D AND I WANT TO 4406 02:41:18,430 --> 02:41:20,732 SHARE THE LEARNINGS WE'VE HAD 4407 02:41:20,732 --> 02:41:22,500 BECAUSE I THINK -- WE'RE VERY 4408 02:41:22,500 --> 02:41:24,035 EXCITED PARTICIPATE IN TRIALS, 4409 02:41:24,035 --> 02:41:27,472 IT WAS REALLY EXCITING TO HAVE 4410 02:41:27,472 --> 02:41:28,273 SOMEONE INTERESTED, WE KNEW 4411 02:41:28,273 --> 02:41:30,375 THERE WOULD BE SOME CHALLENGES 4412 02:41:30,375 --> 02:41:31,643 BUT SOME CHALLENGES WE DIDN'T 4413 02:41:31,643 --> 02:41:32,811 ANTICIPATE AND JUST WANT TO KIND 4414 02:41:32,811 --> 02:41:34,312 OF BRING THOSE TO PEOPLE'S 4415 02:41:34,312 --> 02:41:34,579 ATTENTION. 4416 02:41:34,579 --> 02:41:36,347 SO WE KNEW THERE WOULD BE ISSUES 4417 02:41:36,347 --> 02:41:40,385 WITH RECRUITMENT, IT'S A RARE 4418 02:41:40,385 --> 02:41:43,088 DISEASE, IF YOU LOOK AT THESE 4419 02:41:43,088 --> 02:41:44,589 GENE THESE ARE 56 ULTRA RARE 4420 02:41:44,589 --> 02:41:45,824 DISEASES. 4421 02:41:45,824 --> 02:41:47,225 SO WE ANTICIPATED THAT, WE ALSO 4422 02:41:47,225 --> 02:41:49,561 KNEW THERE WOULD BE ISSUES WITH 4423 02:41:49,561 --> 02:42:00,105 CONFIRMING THE DIAGNOSIS TO GET 4424 02:42:02,140 --> 02:42:05,343 PEOPLE INTO WITH GENETIC TESTINA 4425 02:42:05,343 --> 02:42:08,646 PROBLEM, SO WE NEED MORE TESTING 4426 02:42:08,646 --> 02:42:10,148 AND TO REALLY GOOD 4427 02:42:10,148 --> 02:42:11,583 INTERPRETATION OF TEST RESULTS 4428 02:42:11,583 --> 02:42:12,250 FOR RARE DISEASES. 4429 02:42:12,250 --> 02:42:14,619 WE KNEWHERE WAS GCAL 4430 02:42:14,619 --> 02:42:16,955 DISEASES THAT WOULD BE A PROBLEM 4431 02:42:16,955 --> 02:42:18,256 FOR PARTICIPATION AND WE WERE 4432 02:42:18,256 --> 02:42:20,091 ALSO AWARE OF THE RESEARCH NAIVE 4433 02:42:20,091 --> 02:42:21,126 COMMUNITY THAT REALLY DIDN'T 4434 02:42:21,126 --> 02:42:22,861 HAVE MUCH EXPERIENCE WITH THIS 4435 02:42:22,861 --> 02:42:24,462 AND DIDN'T NECESSARILY 4436 02:42:24,462 --> 02:42:26,464 UNDERSTAND THE IMPORTANCE. 4437 02:42:26,464 --> 02:42:28,633 AND WE KNEW THAT IDEBTIFICATION 4438 02:42:28,633 --> 02:42:32,537 AND THE PATIENT CENSUS PER SITE 4439 02:42:32,537 --> 02:42:33,738 COULD BE CHALLENGING WITH 4440 02:42:33,738 --> 02:42:34,038 RECRUITMENT. 4441 02:42:34,038 --> 02:42:35,840 WHAT WE DID NOT ANTICIPATE 4442 02:42:35,840 --> 02:42:37,609 THAT WE W GET ACTIVE 4443 02:42:37,609 --> 02:42:38,943 RESISTANCE TO CLIRIALSLI BY 4444 02:42:38,943 --> 02:42:41,079 SOME OF OUR SISTER GROUPS IN 4445 02:42:41,079 --> 02:42:43,081 ARBITRATE COUNTRIES AND THAT CAN 4446 02:42:43,081 --> 02:42:43,948 BE DAMAGING GLOBALLY. 4447 02:42:43,948 --> 02:42:48,319 SO THAT WAS A SURPRISE AND AN 4448 02:42:48,319 --> 02:42:49,053 UNPLEASANT SURPRISE. 4449 02:42:49,053 --> 02:42:51,256 AND THEN WE HAD SITE PERFORMANCE 4450 02:42:51,256 --> 02:42:52,223 ISSUES THAT WE HADN'T 4451 02:42:52,223 --> 02:42:53,725 ANTICIPATED AND 1 OF THE BIG 1S 4452 02:42:53,725 --> 02:42:57,162 IN THIS CAME UP EARLIER IN THE 4453 02:42:57,162 --> 02:42:58,229 MEETING TODAY IS CONTRACTING 4454 02:42:58,229 --> 02:42:59,430 DELAYS AND I WILL TELL YOU THAT 4455 02:42:59,430 --> 02:43:00,999 AS WE LOOK AT MOVING INTO OTHER 4456 02:43:00,999 --> 02:43:03,301 TRIALS, WE ARE VERY WELL AWARE 4457 02:43:03,301 --> 02:43:05,370 OF WHICH ACADEMIC CENTERS TOOK 2 4458 02:43:05,370 --> 02:43:07,906 YEARS TO GET PAPERWORK DONE, AND 4459 02:43:07,906 --> 02:43:10,208 THEY ARE NOT REAL,A PEELING WHEN 4460 02:43:10,208 --> 02:43:13,645 WE'RE LOOKING TO PARTNER WITH 4461 02:43:13,645 --> 02:43:14,779 SITES FOR RESEARCH, CLINICAL 4462 02:43:14,779 --> 02:43:15,046 RESEARCH. 4463 02:43:15,046 --> 02:43:17,749 WE HAD A LOT OF TROUBLE WITH THE 4464 02:43:17,749 --> 02:43:20,084 LACK OF COORDINATOR AVAILABILITY 4465 02:43:20,084 --> 02:43:23,154 AND THIS MADE IT VERY SLOW TO 4466 02:43:23,154 --> 02:43:28,693 GET PATIENTS ENROLLED. 4467 02:43:28,693 --> 02:43:31,930 NEXT SLIDE. 4468 02:43:31,930 --> 02:43:33,565 SO SPECIFIC STUDY CHALLENGES, WE 4469 02:43:33,565 --> 02:43:37,101 DID A STUDY ABOUT 7 YEARS AGO 4470 02:43:37,101 --> 02:43:39,871 WITH AN ENAC INHIBITOR, THE 4471 02:43:39,871 --> 02:43:40,605 COMPANY WAS CALLED PARRION AND 4472 02:43:40,605 --> 02:43:44,042 THE STUDY WAS THE CLEAN PC D 4473 02:43:44,042 --> 02:43:47,979 STUDY, THIS WAS AN INHALED ENAC 4474 02:43:47,979 --> 02:43:51,049 INHIBITOR, THE IT T WAS TO PROVE 4475 02:43:51,049 --> 02:43:52,417 AIRWAY CLEARANCE ISSUES THERE 4476 02:43:52,417 --> 02:43:54,886 WERE 2 POSITIVE RESULTS 4477 02:43:54,886 --> 02:43:58,690 PUBLISHED IN LANCET STUDY, THIS 4478 02:43:58,690 --> 02:44:00,425 IS AN OLD STU IT JUST CAME 4479 02:44:00,425 --> 02:44:03,228 OUT THIS YEAR AND THAT'S BECAUSE 4480 02:44:03,228 --> 02:44:04,829 THE COMPANY THAT LICENSED THE 4481 02:44:04,829 --> 02:44:06,931 DRUG PARRION HELD ON TO THE DATA 4482 02:44:06,931 --> 02:44:09,801 AND WE COULD NOT GET IT 4483 02:44:09,801 --> 02:44:10,068 PUBLISHED. 4484 02:44:10,068 --> 02:44:11,936 SO RIGHT NOW THERE'S PHASE 3 4485 02:44:11,936 --> 02:44:13,238 PLANS BUT FUNDER NEEDS TO BE 4486 02:44:13,238 --> 02:44:15,540 FOUND TO COMPLETE THIS TRIAL. 4487 02:44:15,540 --> 02:44:17,508 IT REQUIRED MORE THAN 30 SITES 4488 02:44:17,508 --> 02:44:22,180 AND 8 COUNTRIES TO COMPLETE 4489 02:44:22,180 --> 02:44:23,548 ENROLLMENT OF THE PATIENTS, 4490 02:44:23,548 --> 02:44:25,183 AGAIN THIS WAS 7 YEARS AGO SO WE 4491 02:44:25,183 --> 02:44:27,952 MAY NOT BE FACING THE SAME 4492 02:44:27,952 --> 02:44:28,253 CHALLENGES. 4493 02:44:28,253 --> 02:44:30,388 SOME SITES DID NOT EVEN GET 4494 02:44:30,388 --> 02:44:32,123 THE-HAD FINISH THEIR CONTRACTING 4495 02:44:32,123 --> 02:44:34,559 STUFF THROUGH THEIR ACADEMIC 4496 02:44:34,559 --> 02:44:35,727 SITES UNTIL AFTER THE STUDY 4497 02:44:35,727 --> 02:44:38,363 CLOSED SO THEY NEVER EVEN GOT A 4498 02:44:38,363 --> 02:44:40,265 PATIENT IN, AND FOR AN INDUSTRY 4499 02:44:40,265 --> 02:44:41,332 PARTNER, THERE'S A OHE MONEY 4500 02:44:41,332 --> 02:44:46,304 THAT GOES INTO BRINGS SITES ON 4501 02:44:46,304 --> 02:44:47,438 BOARD SO SITE SELECTION IS 4502 02:44:47,438 --> 02:44:47,805 CRUCIAL. 4503 02:44:47,805 --> 02:44:49,340 THE STUDY DESIGN WAS 4504 02:44:49,340 --> 02:44:50,174 CHALLENGING, WE WERE INVOLVED IN 4505 02:44:50,174 --> 02:44:51,609 HELPING WITH THE STUDY DESIGN 4506 02:44:51,609 --> 02:44:53,211 BUT THE THINGS THAT WE FELT 4507 02:44:53,211 --> 02:44:55,947 WOULD MAKE IT DIFFICULT FOR 4508 02:44:55,947 --> 02:44:57,115 PATIENTS TO PARTICIPATE WERE 4509 02:44:57,115 --> 02:44:59,517 OVERRULED BY THE NEEDS OF THE 4510 02:44:59,517 --> 02:45:01,219 REGULATOR AND THAT ALSO CREATED 4511 02:45:01,219 --> 02:45:01,786 SOME CHALLENGES. 4512 02:45:01,786 --> 02:45:04,422 SO WE LEARNED CAREFUL SITE 4513 02:45:04,422 --> 02:45:07,825 SELECTION IS CRUCIAL AND THAT 4514 02:45:07,825 --> 02:45:09,327 ALL FUTURE PC D CLINICAL TRIALS 4515 02:45:09,327 --> 02:45:11,863 ARE GOING TO REQUIRE 4516 02:45:11,863 --> 02:45:12,664 INTERNATIONAL COLLABORATION 4517 02:45:12,664 --> 02:45:13,932 BECAUSE THERE JUST AREN'T ENOUGH 4518 02:45:13,932 --> 02:45:21,239 PATIENTS IN ANY 1 REGION. 4519 02:45:21,239 --> 02:45:22,840 NEXT SLIDE. 4520 02:45:22,840 --> 02:45:24,108 SO WE'RE CURRENTLY INVOLVED WITH 4521 02:45:24,108 --> 02:45:28,713 PHASE 1 B STUDIES THIS IS THE 4522 02:45:28,713 --> 02:45:30,348 NOVEL INHALED MRNA THERAPY AND 4523 02:45:30,348 --> 02:45:33,117 IT'S DESIGNED TO REPAIR THE 4524 02:45:33,117 --> 02:45:34,519 UNDERLYING DEFECT OR AT LEAST 4525 02:45:34,519 --> 02:45:36,821 REPAIR THE MESSAGING FOR THE 4526 02:45:36,821 --> 02:45:39,257 UNDERLYING DEFECT IN PC D THAT'S 4527 02:45:39,257 --> 02:45:41,492 CAUSED IN VARIANTS IN DNAI1. 4528 02:45:41,492 --> 02:45:43,494 THERE ARE SITES IN THE UK, 4529 02:45:43,494 --> 02:45:47,432 GERMANY AND DENMARK IN ADDITION 4530 02:45:47,432 --> 02:45:48,967 TO US, AND BECAUSE OF THE 4531 02:45:48,967 --> 02:45:53,938 DIAGNOSTIC ISSUES AND THE 4532 02:45:53,938 --> 02:45:56,374 SPECIFICALLY FOR DNAI1, THE SITE 4533 02:45:56,374 --> 02:45:58,142 IS STUDYING TESTING FOR ADULTS 4534 02:45:58,142 --> 02:46:00,078 WITH PC D, UPTAKE ON THIS AS 4535 02:46:00,078 --> 02:46:01,012 BEEN SLOWER THAN ANTICIPATED AND 4536 02:46:01,012 --> 02:46:03,081 PART OF THIS IS BECAUSE THE 4537 02:46:03,081 --> 02:46:05,783 CLINICIANS THAT ARE INVOLVED ARE 4538 02:46:05,783 --> 02:46:08,319 A LITTLE AMBIVALENT ABOUT IT, 4539 02:46:08,319 --> 02:46:10,154 IT'S BEEN CHALLENGING TO FIND 4540 02:46:10,154 --> 02:46:12,724 9-10 PATIENTS WHO FIT THE 4541 02:46:12,724 --> 02:46:14,258 INCLUSION CRITERIA AND ARE ABLE 4542 02:46:14,258 --> 02:46:16,894 TO TRAVEL AND SPEND A WEEK ATLAS 4543 02:46:16,894 --> 02:46:18,529 VEGAS AT THE CRO. 4544 02:46:18,529 --> 02:46:21,299 THERE IS SIGNIFICANT INTEREST IN 4545 02:46:21,299 --> 02:46:22,000 PARTICIPATING EXPRESSED BY 4546 02:46:22,000 --> 02:46:23,768 PATIENTS BUT WE HAVE DONE 4547 02:46:23,768 --> 02:46:24,836 SURVEYS MANY TIMES TO FIND OUT 4548 02:46:24,836 --> 02:46:27,905 WHAT KIND OF BARRIERS THERE ARE 4549 02:46:27,905 --> 02:46:29,507 TO PARTICIPATION AND PATIENTS 4550 02:46:29,507 --> 02:46:33,044 ALWAYS ARE FAR MORE WILLING TO 4551 02:46:33,044 --> 02:46:34,645 PARTICIPATE IN GENERAL THAN WHEN 4552 02:46:34,645 --> 02:46:36,848 THE SPECIFIC REQUIREMENTS OF THE 4553 02:46:36,848 --> 02:46:37,281 STUDY COMES OUT. 4554 02:46:37,281 --> 02:46:38,850 I DO ALSO WANT TO MAKE A POINT 4555 02:46:38,850 --> 02:46:40,685 HERE THAT SOMETIMES WE HEAR THAT 4556 02:46:40,685 --> 02:46:42,754 THEY DON'T WANT TO BE 4557 02:46:42,754 --> 02:46:44,222 INCONVENIENCED, I DON'T THINK 4558 02:46:44,222 --> 02:46:47,725 THAT'S TRUE. 4559 02:46:47,725 --> 02:46:50,461 IT'S NOT A CONVENIENCE ISSUE, 4560 02:46:50,461 --> 02:46:52,397 IT'S THAT THEY'RE LITERALLY 4561 02:46:52,397 --> 02:46:53,197 JEOPARDIZING THEIR EDUCATION, 4562 02:46:53,197 --> 02:46:54,432 JOBS SO THERE'S A LOT OF THINGS 4563 02:46:54,432 --> 02:46:55,500 PATIENTS HAVE TO THINK ABOUT 4564 02:46:55,500 --> 02:46:57,869 BEFORE THEY COMMIT TO A CLINICAL 4565 02:46:57,869 --> 02:46:58,669 TRIAL AND THE MODEL CITIZENNER 4566 02:46:58,669 --> 02:47:02,573 WE DID DO TO MAKE CLINICAL TRIAL 4567 02:47:02,573 --> 02:47:04,308 STUDY DESIGN ACCOMMODATE THESE 4568 02:47:04,308 --> 02:47:07,745 THINGS, THE BETTER IT'LL BE TO 4569 02:47:07,745 --> 02:47:08,646 RECRUIT INTO TRIALS. 4570 02:47:08,646 --> 02:47:10,515 SO AGAIN WE LEARN THAT SITE 4571 02:47:10,515 --> 02:47:10,982 SELECTION IS CRUCIAL. 4572 02:47:10,982 --> 02:47:12,450 YOU HAVE TO HAVE THE RIGHT SITES 4573 02:47:12,450 --> 02:47:14,052 THAT ARE MOTIVATED AND WILL FIND 4574 02:47:14,052 --> 02:47:15,887 THE PATIENTS AND THAT AGAIN, 4575 02:47:15,887 --> 02:47:18,689 WE'RE GOING TO NEED 4576 02:47:18,689 --> 02:47:24,328 INTERNATIONAL COLLABORATION. 4577 02:47:24,328 --> 02:47:24,662 NEXT SLIDE. 4578 02:47:24,662 --> 02:47:27,031 ONE OF THE THINGS WE DID TO HELP 4579 02:47:27,031 --> 02:47:27,732 OVERCOME THESE CHALLENGES, WE 4580 02:47:27,732 --> 02:47:30,935 DID A SERIES OF WEBINARS WITH 4581 02:47:30,935 --> 02:47:34,539 RICO TO TALK ABOUT GENETICS, 4582 02:47:34,539 --> 02:47:36,941 GENETIC BASE THERAPIES AND ABOUT 4583 02:47:36,941 --> 02:47:39,644 THE THERAPY THAT RECODE HAD ON 4584 02:47:39,644 --> 02:47:41,179 OFFER, THESE WERE WELL ATTENDED. 4585 02:47:41,179 --> 02:47:43,147 WE HAD CLOSE TO 200 PEOPLE AT 4586 02:47:43,147 --> 02:47:44,782 EACH OF THESE WEBINARS AND WE 4587 02:47:44,782 --> 02:47:47,518 DID PICK UP SOME PATIENTS FOR 4588 02:47:47,518 --> 02:47:49,954 RECRUITMENT THROUGH DOING THIS 4589 02:47:49,954 --> 02:47:57,562 JOINT EDUCATIONAL ACTIVITY. 4590 02:47:57,562 --> 02:47:58,429 NEXT SLIDE. 4591 02:47:58,429 --> 02:48:00,765 WE ALSO HELD OUR FIRST EVER 4592 02:48:00,765 --> 02:48:03,601 GLOBAL PATIENT ADVOCACY SUMMIT 4593 02:48:03,601 --> 02:48:05,236 IN LONDON IN 2024 TO GET 4594 02:48:05,236 --> 02:48:06,871 EVERYBODY ALIGNED ON MESSAGING 4595 02:48:06,871 --> 02:48:09,373 ABOUT THE IMPORTANCE OF CLINICAL 4596 02:48:09,373 --> 02:48:10,942 TRIALS. 4597 02:48:10,942 --> 02:48:12,243 IT WAS VERY INTERESTING, I THINK 4598 02:48:12,243 --> 02:48:14,912 IT WAS SHOCKING TO ME, HOW 4599 02:48:14,912 --> 02:48:16,347 POORLY RESOURCED SOME OF THESE 4600 02:48:16,347 --> 02:48:17,982 GROUPS ARE, HOW WILLING AND 4601 02:48:17,982 --> 02:48:19,350 DEDICATED AND COMMITTED THEY ARE 4602 02:48:19,350 --> 02:48:21,419 BUT THEY REALLY HAVE VERY LITTLE 4603 02:48:21,419 --> 02:48:26,991 TO WORK WITH. 4604 02:48:26,991 --> 02:48:29,060 NEXT SLIDE. 4605 02:48:29,060 --> 02:48:31,596 SO OUR NEXT PLAN TO OVERCOME 4606 02:48:31,596 --> 02:48:33,698 CHALLENGES IS AGAIN, TO BORROW 4607 02:48:33,698 --> 02:48:35,500 FROM THE CYSTIC FIBROSIS 4608 02:48:35,500 --> 02:48:35,967 FOUNDATION. 4609 02:48:35,967 --> 02:48:37,969 THEY HAVE HAD TREMENDOUS SUCCESS 4610 02:48:37,969 --> 02:48:39,370 WITH THEIR THERAPEUTICS 4611 02:48:39,370 --> 02:48:40,538 DEVELOPMENT NETWORK, WE ARE 4612 02:48:40,538 --> 02:48:42,273 TRYING TO LAUNCH WHAT WE CALL 4613 02:48:42,273 --> 02:48:43,374 THE CLINICAL NETWORK FOR TRIALS, 4614 02:48:43,374 --> 02:48:46,511 WHICH WILL BE A VERY MINISCALED 4615 02:48:46,511 --> 02:48:48,412 DOWN VERSION OF THAT, BUT WE'RE 4616 02:48:48,412 --> 02:48:52,116 ATTEMPTING TO CREATE A 1 STOP 4617 02:48:52,116 --> 02:48:54,118 SHOP FOR INDUSTRY PARTNERS AND 4618 02:48:54,118 --> 02:48:55,553 DEVELOPERS AND VENTURE C 4619 02:48:55,553 --> 02:48:57,054 APITALISTS SO THAT THERE'S 1 4620 02:48:57,054 --> 02:48:58,589 POINT OF CONTACT AND FROM THAT 4621 02:48:58,589 --> 02:48:59,991 THEY WILL GET INTO OUR NETWORK. 4622 02:48:59,991 --> 02:49:03,127 THIS IS KIND OF OUR 4623 02:49:03,127 --> 02:49:06,197 SUSTAINABILITY PLAN FOR THE 4624 02:49:06,197 --> 02:49:09,767 CONTINUED ACTIVITIES OF THE 4625 02:49:09,767 --> 02:49:11,502 GENETIC DISORDERS OF MUCUS 4626 02:49:11,502 --> 02:49:14,038 SILLIARY CONSORTIUM AND WATCH 4627 02:49:14,038 --> 02:49:14,906 THIS SPACE, HOPE EMPLOYYLY WE 4628 02:49:14,906 --> 02:49:17,074 WILL BE VERY SUCCESSFUL WITH 4629 02:49:17,074 --> 02:49:17,308 THAT. 4630 02:49:17,308 --> 02:49:26,717 I THINK THAT THAT'S IT FOR ME. 4631 02:49:26,717 --> 02:49:29,820 THANK YOU. 4632 02:49:29,820 --> 02:49:30,621 >> THANK YOU MICHELE. 4633 02:49:30,621 --> 02:49:33,357 SO WE WILL MOVE INTO OUR NEXT 4634 02:49:33,357 --> 02:49:43,868 SPEAKER WHICH IS DR. NICKERSON 4635 02:49:46,604 --> 02:49:46,771 NUTTER. 4636 02:49:46,771 --> 02:49:49,340 >> GREAT, I WANT TO THANK THE 4637 02:49:49,340 --> 02:49:50,441 ORGANIZERS FOR THE OPPORTUNITY 4638 02:49:50,441 --> 02:49:51,976 TO SPEAK TO YOU TODAY ABOUT WHAT 4639 02:49:51,976 --> 02:49:53,077 WE'RE DOING AT 3 LAKES 4640 02:49:53,077 --> 02:49:53,411 FOUNDATION. 4641 02:49:53,411 --> 02:49:55,146 IT WAS A PRIVATE FOUNDATION 4642 02:49:55,146 --> 02:49:57,815 STARTED BY A FAMILY WHO LOST A 4643 02:49:57,815 --> 02:49:59,283 FAMILY MEMBER TO PULMONARY 4644 02:49:59,283 --> 02:50:00,318 FIBROSIS, SO WE'RE A BIT 4645 02:50:00,318 --> 02:50:01,485 DIFFERENT FROM THE OTHER 4646 02:50:01,485 --> 02:50:03,221 FOUNDATIONS IN THAT WE'RE NOT 4647 02:50:03,221 --> 02:50:07,959 FOCUSED SOLELY ON ADVOCACY, 4648 02:50:07,959 --> 02:50:08,926 ADVOCACY IS IMPORTANT BUT 4649 02:50:08,926 --> 02:50:10,194 LUCKILY THERE ARE OTHER 4650 02:50:10,194 --> 02:50:12,263 FOUNDATIONS SUCH AS THE 4651 02:50:12,263 --> 02:50:14,232 PULMONARY FOUNDATION AND PF 4652 02:50:14,232 --> 02:50:15,499 WARRIORS WHO ARE AVAILABLE TO 4653 02:50:15,499 --> 02:50:17,535 PATIENTS AND HAVE GOOD ADVOCACY 4654 02:50:17,535 --> 02:50:17,969 FOR PATIENTS. 4655 02:50:17,969 --> 02:50:20,271 SO OUR NICHE IS WE'RE MORE 4656 02:50:20,271 --> 02:50:23,608 FOCUSED ON THE SCIENCE TO TRY TO 4657 02:50:23,608 --> 02:50:25,509 ADVANCE THERAPIES AND ALSO 4658 02:50:25,509 --> 02:50:31,282 UNDERSTAND EARLY DISEASE AND 4659 02:50:31,282 --> 02:50:31,849 ACCELERATE DIAGNOSIS FOR 4660 02:50:31,849 --> 02:50:32,850 PATIENTS. 4661 02:50:32,850 --> 02:50:39,090 SO THE NEXT SLIDE. 4662 02:50:39,090 --> 02:50:41,192 SO SINCE WE'RE COVERING A LOT OF 4663 02:50:41,192 --> 02:50:42,260 DIFFERENT DECS IN THIS WORKSHOP, 4664 02:50:42,260 --> 02:50:46,631 I WANT TO BRIEFLY GO OVER IDIO 4665 02:50:46,631 --> 02:50:47,231 PATHIC PULMONARY FIBROSIS, SO 4666 02:50:47,231 --> 02:50:49,567 THIS IS WHAT THE FAMILY I'M 4667 02:50:49,567 --> 02:50:51,068 WORKING WITH ENCOUNTERED AND 4668 02:50:51,068 --> 02:50:53,337 THEY HAD CHALLENGES WITH GETTING 4669 02:50:53,337 --> 02:50:55,339 AN ACCURATE DIAGNOSIS WHICH IS A 4670 02:50:55,339 --> 02:50:59,410 COMMON STORY AND THEY ALSO HAD 4671 02:50:59,410 --> 02:51:01,145 TROUBLE WITH OXYGEN AND THEY 4672 02:51:01,145 --> 02:51:02,713 WERE NOT VERY GOOD EFFECTIVE 4673 02:51:02,713 --> 02:51:03,214 THERAPIES. 4674 02:51:03,214 --> 02:51:05,516 THIS WAS ABOUT 10 YEARS AGO. 4675 02:51:05,516 --> 02:51:06,450 SO, FOR ANYBODY WHO'S 4676 02:51:06,450 --> 02:51:09,387 EXPERIENCED IT, IT'S A CHRONIC 4677 02:51:09,387 --> 02:51:10,021 AND PROGRESSIVE DISEASE WHERE 4678 02:51:10,021 --> 02:51:12,790 YOU KNOW YOU LOSE THE ABILITY TO 4679 02:51:12,790 --> 02:51:14,158 BREATHE, IT'S A RARE DISEASE, 4680 02:51:14,158 --> 02:51:16,127 IT'S A RARE DISEASE ABOUT 2 4681 02:51:16,127 --> 02:51:18,462 HELPED THOUSAND PATIENTS IN THEP 4682 02:51:18,462 --> 02:51:22,533 U.S. AND WHEN PATIENTS ARE 4683 02:51:22,533 --> 02:51:23,668 DIAGNOSED, THEY'RE OFTEN TOLD, 4684 02:51:23,668 --> 02:51:26,437 YOU KNOW YOU HAVERY 4685 02:51:26,437 --> 02:51:27,471 FIBROSIS, SOME PATIENTS MAY NOT 4686 02:51:27,471 --> 02:51:29,540 EVEN KNOW WHAT THIS IS AND GO 4687 02:51:29,540 --> 02:51:30,941 GET YOUR AFFAIRS IN ORDER 4688 02:51:30,941 --> 02:51:34,011 BECAUSE YOU CAN LIVE 2-5 YEARS. 4689 02:51:34,011 --> 02:51:37,682 THIS CAN BE OBVIOUSLY 4690 02:51:37,682 --> 02:51:37,982 DEVASTATING. 4691 02:51:37,982 --> 02:51:40,318 THERE'S ABOUT 30,000 DEATHS PER 4692 02:51:40,318 --> 02:51:45,823 YEAR, IT ALSO HAS STRONG GENETIC 4693 02:51:45,823 --> 02:51:46,657 COMPONENT OR FAMILIAL COMPONENT 4694 02:51:46,657 --> 02:51:52,697 SO IF HAVE YOU A PARENT WHO HAST 4695 02:51:52,697 --> 02:51:53,831 6-10 TIMES MORE LIKELY TO HAVE 4696 02:51:53,831 --> 02:51:56,367 IT THAN THE GENERAL POPULATION 4697 02:51:56,367 --> 02:51:59,737 AND DIAGNOSIS OFTEN IT'S ON NOT 4698 02:51:59,737 --> 02:52:02,506 SO MUCH A BIOPSY ANYMORE BUT A 4699 02:52:02,506 --> 02:52:05,676 CT SCAN OR HIGH RESOLUTION CT 4700 02:52:05,676 --> 02:52:07,445 SCAN AND OFTEN BY THE TIME YOU 4701 02:52:07,445 --> 02:52:08,579 GOET TO THE PULL 4702 02:52:08,579 --> 02:52:10,548 MONITORROLOGYIST FOR THE CT 4703 02:52:10,548 --> 02:52:13,684 SCAN, YOU'VE ALREADY LOST A LOT 4704 02:52:13,684 --> 02:52:15,619 OF LUNG FUNCTION AND YOUR 4705 02:52:15,619 --> 02:52:17,588 ABILITY TO BREATHE IS ALREADY 4706 02:52:17,588 --> 02:52:18,723 GOING DOWN. 4707 02:52:18,723 --> 02:52:20,791 SO THE TREATMENTS THERE ARE 2 4708 02:52:20,791 --> 02:52:24,295 APPROVED TREATMENTS, THESE WERE 4709 02:52:24,295 --> 02:52:34,572 BOTH APPROVED IN 2014, NINTADA 4710 02:52:34,572 --> 02:52:35,539 NIB AND PIRFENIDONE, IT'S GREAT 4711 02:52:35,539 --> 02:52:37,408 WE HAVE THESE MEDICATIONS BUT 4712 02:52:37,408 --> 02:52:38,909 THERE ARE SEERSZ SIDE EFFECTS 4713 02:52:38,909 --> 02:52:40,845 AND SOME PATES FIND THEM 4714 02:52:40,845 --> 02:52:42,613 INTOLERABLE AND THE OTHER OPTION 4715 02:52:42,613 --> 02:52:50,488 IS LUNG TRANSPLANTATION. 4716 02:52:50,488 --> 02:52:51,021 SO NEXT SLIDE. 4717 02:52:51,021 --> 02:52:54,925 SO I JUST WANTED TO TALK ABOUT 4718 02:52:54,925 --> 02:52:57,094 THE DRUG TIMELINE OF PULMONARY 4719 02:52:57,094 --> 02:52:59,797 FIBROSIS, WHERE WE'VE BEEN AND 4720 02:52:59,797 --> 02:53:03,134 WHERE WE'RE AT. 4721 02:53:03,134 --> 02:53:04,402 SO PERFENIDONE WAS DISCOVERED IN 4722 02:53:04,402 --> 02:53:08,606 1976 BUT IT WASN'T STUDY AND FOR 4723 02:53:08,606 --> 02:53:09,673 THE FIBROTIC PROPERTIES, SO IT 4724 02:53:09,673 --> 02:53:10,841 WAS MORE FOR INFLAMMATION AND 4725 02:53:10,841 --> 02:53:14,912 THEN IT WAS TESTED IN A 4726 02:53:14,912 --> 02:53:16,380 BLEOMYCIN O HAMSTERS WHERE IT 4727 02:53:16,380 --> 02:53:19,016 WAS FOUND TO HAVE ANTIFIBROTIC 4728 02:53:19,016 --> 02:53:19,583 PROPERTIES. 4729 02:53:19,583 --> 02:53:24,088 THERE WAS A CONTROL TRIAL IN 4730 02:53:24,088 --> 02:53:25,756 JAPAN, THAT WAS DISCONTINUED IN 4731 02:53:25,756 --> 02:53:30,628 2005, BUT THE USE FOR 4732 02:53:30,628 --> 02:53:32,496 PERFENIDONE WAS ACCEPTED THEN. 4733 02:53:32,496 --> 02:53:36,400 THERE WERE STUDIES THAT SHOWED 4734 02:53:36,400 --> 02:53:38,736 EFFECT ON FVC, SORRY, IN 2011 4735 02:53:38,736 --> 02:53:41,205 AND THEN BASED ON THE CAPACITY 4736 02:53:41,205 --> 02:53:43,073 STUDY AND ANOTHER STUDY CALLED 4737 02:53:43,073 --> 02:53:47,278 THE ASCEND STUDY, THE DRUG WAS 4738 02:53:47,278 --> 02:53:50,748 WAS APPROVE INDEED 2014. 4739 02:53:50,748 --> 02:53:51,949 FOR NINTEDANIB, THE PATH WAS A 4740 02:53:51,949 --> 02:53:52,716 BIT SHORTER. 4741 02:53:52,716 --> 02:53:55,953 THE DRUG WAS DEVELOPED -- THE 4742 02:53:55,953 --> 02:53:58,122 NUMBER FOR THIS DRUG WAS BI1120, 4743 02:53:58,122 --> 02:54:02,193 AND IT WAS SHOWN TO HAVE EFFECT 4744 02:54:02,193 --> 02:54:04,662 ON LEUKEMIC CELL ASSAYS TO 4745 02:54:04,662 --> 02:54:06,363 COLONY FOR THE PURPOSING UNIT 4746 02:54:06,363 --> 02:54:10,568 ANDS THERE WAS WAS AN AN LOG 4747 02:54:10,568 --> 02:54:12,403 DRUG CALLED BI1000 WHICH WAS 4748 02:54:12,403 --> 02:54:14,004 SHOWN TO HAVE ANTIFIBROTIC 4749 02:54:14,004 --> 02:54:18,776 PROPERTIES IN A BLEO MOUSE 4750 02:54:18,776 --> 02:54:19,076 MODEL. 4751 02:54:19,076 --> 02:54:21,645 SO THEN THERE WAS A PHASE 1 4752 02:54:21,645 --> 02:54:24,715 STUDY WHERE SAWAWHE FDC 4753 02:54:24,715 --> 02:54:26,183 DECLINE IN 2010 AND THERE WERE A 4754 02:54:26,183 --> 02:54:28,252 A OTHER STUDIES CALLED THE 4755 02:54:28,252 --> 02:54:31,055 IMPULSEUS STUDIES WHERE THEY 4756 02:54:31,055 --> 02:54:33,290 SHOWED THE EFFECTS ON FVC WHICH 4757 02:54:33,290 --> 02:54:37,094 WERE THE BASIS FOR APPROVAL IN 4758 02:54:37,094 --> 02:54:44,268 2014. 4759 02:54:44,268 --> 02:54:45,035 NEXT SLIDE, PLEASE. 4760 02:54:45,035 --> 02:54:49,940 SO THE GOOD THING WAS THAT THE 4761 02:54:49,940 --> 02:54:51,208 DRUGS REALLY DISPLAYED THE 4762 02:54:51,208 --> 02:54:53,477 IMPROVEMENT OF THE DRUGS WITH AN 4763 02:54:53,477 --> 02:54:54,411 APPROVAL PATHWAY. 4764 02:54:54,411 --> 02:54:56,080 HERE WAS THE DRUG PIPELINE IN 4765 02:54:56,080 --> 02:54:56,313 2021. 4766 02:54:56,313 --> 02:55:00,618 THERE WAS A LOT OF WORK BETWEEN 4767 02:55:00,618 --> 02:55:02,319 2014-2021 AND THESE WERE ALL PUT 4768 02:55:02,319 --> 02:55:03,687 INTO THE PIPELINE WITH DIFFERENT 4769 02:55:03,687 --> 02:55:04,622 MECHANISMS, AND THERE WAS A LOT 4770 02:55:04,622 --> 02:55:06,924 OF HOPE FOR PATIENTS 4771 02:55:06,924 --> 02:55:07,825 UNFORTUNATELY, NONE OF THESE 4772 02:55:07,825 --> 02:55:11,595 HAVE MADE IT ALL THE WAY TO THE 4773 02:55:11,595 --> 02:55:12,029 APPROVAL STAGE. 4774 02:55:12,029 --> 02:55:14,064 SO WHY IS THAT? 4775 02:55:14,064 --> 02:55:24,608 SO YOU CAN GO TO THE NEXT SLIDE. 4776 02:55:25,743 --> 02:55:27,711 THIS JUST SHOWS HOW MANY WE 4777 02:55:27,711 --> 02:55:27,945 LOST. 4778 02:55:27,945 --> 02:55:29,079 I UPDATED IT THIS YEAR. 4779 02:55:29,079 --> 02:55:32,416 SO CAN YOU SEE AT THE PHASE 3 WE 4780 02:55:32,416 --> 02:55:33,851 LOST ABOUT HALF, WE LOST HALF OF 4781 02:55:33,851 --> 02:55:35,286 THE PHASE 2 AND JUST 1 OF THE 4782 02:55:35,286 --> 02:55:36,120 PHASE 1. 4783 02:55:36,120 --> 02:55:37,254 SO IT'S INTERESTING BECAUSE 4784 02:55:37,254 --> 02:55:39,890 PHASE 1 IS OFTEN SAFETY AND YOU 4785 02:55:39,890 --> 02:55:41,992 CAN SEE THAT MAYBE WE'RE DOING 4786 02:55:41,992 --> 02:55:43,661 BETTER ON SAFETY, GETTING PAST 4787 02:55:43,661 --> 02:55:46,630 THE PHASE 1 STAGE BUT WE'RE 4788 02:55:46,630 --> 02:55:47,565 LOSING THESE BASED ON EFFICACY 4789 02:55:47,565 --> 02:55:50,134 FOR A LOT OF THEM. 4790 02:55:50,134 --> 02:55:51,669 SO WHAT'S GOING ON, RIGHT? 4791 02:55:51,669 --> 02:55:53,404 BECAUSE CLEARLY WE HAD A 4792 02:55:53,404 --> 02:55:55,039 PRECLINICAL PACKAGE WHICH 4793 02:55:55,039 --> 02:55:57,007 DEMONSTRATED EFFICACY BUT THE 4794 02:55:57,007 --> 02:55:59,543 PROBLEM IS, THESE ARE BASED A 4795 02:55:59,543 --> 02:56:03,280 LOT OF TIMES ON CELL BASED 4796 02:56:03,280 --> 02:56:08,152 ASSAYS, AND THEN ON RODENT 4797 02:56:08,152 --> 02:56:09,520 MODELS, MAINLY BLEOMYCIN MODELS 4798 02:56:09,520 --> 02:56:12,056 AND AS WAS DISCUSSED YESTERDAY, 4799 02:56:12,056 --> 02:56:13,223 THE RODENT LUNGS ARE VERY 4800 02:56:13,223 --> 02:56:14,091 DIFFERENT THAN THE HUMAN LUNG 4801 02:56:14,091 --> 02:56:16,193 ANDS MAY NOT BE REPRESENTATIVE 4802 02:56:16,193 --> 02:56:19,730 SO WE REALLY, WE NEED TO DO 4803 02:56:19,730 --> 02:56:21,098 BETTER THAN THIS. 4804 02:56:21,098 --> 02:56:31,342 SO 1 WAY, -- OH, SO THE NEXT 4805 02:56:31,342 --> 02:56:31,976 SLIDE, PLEASE. 4806 02:56:31,976 --> 02:56:33,744 SO THIS JUST SHOWS THE 1S THAT 4807 02:56:33,744 --> 02:56:34,912 HAVE DROPPED OFF AND THEN YOU 4808 02:56:34,912 --> 02:56:38,349 CAN JUST GO TO THE NEXT 1 WHICH 4809 02:56:38,349 --> 02:56:46,390 SHOWS THE CURRENT PIPELINE. 4810 02:56:46,390 --> 02:56:48,859 SO THE GOOD NEWS IS WE HAVE A 4811 02:56:48,859 --> 02:56:50,160 FULL PIPELINE BUT AGAIN NOT THAT 4812 02:56:50,160 --> 02:56:51,729 MANY ARE MAKING IT ALL THE WAY 4813 02:56:51,729 --> 02:56:53,897 AND RIGHT NOW, WE DO HAVE SOME 4814 02:56:53,897 --> 02:56:56,700 PROMISING DRUGS IN PHASE 3, 4815 02:56:56,700 --> 02:57:00,804 THERE'S A PD4 INHIBITOR, FROM VI 4816 02:57:00,804 --> 02:57:05,509 IS AN LPA INHIBITOR FROM BMS, 4817 02:57:05,509 --> 02:57:07,678 THERE'S ANOTHER PROMISING 4818 02:57:07,678 --> 02:57:10,314 THERAPY FOR TGF BETA COMING OUT 4819 02:57:10,314 --> 02:57:11,815 OF [INDISCERNIBLE], SO HOPEFULLY 4820 02:57:11,815 --> 02:57:13,417 SOME OF THESE WILL MAKE IT BUT 4821 02:57:13,417 --> 02:57:15,653 AGAIN THESE HAVE BEEN DEVELOPED, 4822 02:57:15,653 --> 02:57:16,253 THEY'RE PRECLINICAL PACKAGE, 4823 02:57:16,253 --> 02:57:18,856 THEY'RE A BIT OLDER SO THEY MAY 4824 02:57:18,856 --> 02:57:23,093 STILL BE BASED ON BLEOMYCIN I 4825 02:57:23,093 --> 02:57:25,596 KNOW THAT A CLIENT AS SOME 4826 02:57:25,596 --> 02:57:26,563 PRECISION LUNG CUT DATA WHICH I 4827 02:57:26,563 --> 02:57:37,074 WILL TALK ABOUT IN A MINUTE. 4828 02:57:37,074 --> 02:57:37,708 NEXT SLIDE. 4829 02:57:37,708 --> 02:57:42,146 SO IN ORDER TO DO BETTER WE NEED 4830 02:57:42,146 --> 02:57:43,514 TO GET BETTER MODELS AND 4831 02:57:43,514 --> 02:57:44,048 RESEARCH TOOLS. 4832 02:57:44,048 --> 02:57:46,850 SO 2 YEARS AGO THERE WAS AN 4833 02:57:46,850 --> 02:57:49,420 NHLBI WORKSHOP THAT 3 LAKES 4834 02:57:49,420 --> 02:57:51,055 PULMONARY FIBROSIS FOUNDATION 4835 02:57:51,055 --> 02:57:52,423 AND NHLBI SPONSORED WHERE WE 4836 02:57:52,423 --> 02:57:55,159 BROUGHT TOGETHER EXPERTS IN THE 4837 02:57:55,159 --> 02:57:56,393 FIELD AND REALLY ASKED WHAT DO 4838 02:57:56,393 --> 02:57:59,797 WE NEED TO DO TO IMPROVE THE 4839 02:57:59,797 --> 02:58:01,398 DRUG PIPELINE AND GET THERAPIES 4840 02:58:01,398 --> 02:58:01,832 TO PATIENTS. 4841 02:58:01,832 --> 02:58:05,469 AND SO THIS IS WHAT WAS 4842 02:58:05,469 --> 02:58:05,836 IDENTIFIED. 4843 02:58:05,836 --> 02:58:07,805 WE NEED NOVEL MODELS, IT WOULD 4844 02:58:07,805 --> 02:58:10,941 BE GREAT TO HAVE THEM BASED ON 4845 02:58:10,941 --> 02:58:13,711 HUMAN TISSUE, AND ALSO, CAN WE 4846 02:58:13,711 --> 02:58:15,446 HAVE A CENTRAL REPOSITORY FOR 4847 02:58:15,446 --> 02:58:16,547 SINGLE CELL ANALYSIS OF TISSUES 4848 02:58:16,547 --> 02:58:18,048 AND MODELS AND I WILL MENTION A 4849 02:58:18,048 --> 02:58:20,718 LITTLE BIT ABOUT THE IPF CELL 4850 02:58:20,718 --> 02:58:22,786 ATLAS AND THE NEXT SLIDE THAT 3 4851 02:58:22,786 --> 02:58:25,823 LAKES WAS A PART OF. 4852 02:58:25,823 --> 02:58:29,193 ALSO TO UNDERSTAND EARLY DISEASE 4853 02:58:29,193 --> 02:58:30,861 IN WAYS TO IMPROVE DIAGNOSIS, TO 4854 02:58:30,861 --> 02:58:32,563 UNDERSTAND RISK FACTORS AND THE 4855 02:58:32,563 --> 02:58:34,865 DRIVERS, YOU KNOW IF WE CAN 4856 02:58:34,865 --> 02:58:36,567 UNDERSTAND THE DISEASE EARLIER 4857 02:58:36,567 --> 02:58:39,336 WE CAN BACK UP TREATMENT, MAYBE 4858 02:58:39,336 --> 02:58:41,138 WE CAN PREVENT PEOPLE FROM 4859 02:58:41,138 --> 02:58:42,372 GETTING SO FIBROTIC. 4860 02:58:42,372 --> 02:58:44,007 AND ALSO INPOETICIVATIVE 4861 02:58:44,007 --> 02:58:45,109 APPROACHES TO INNOVATIVE TRIAL 4862 02:58:45,109 --> 02:58:45,342 DESIGN. 4863 02:58:45,342 --> 02:58:47,878 WE REALLY NEED TO START, WE NEED 4864 02:58:47,878 --> 02:58:49,546 BETTER END POINTS AS WE'VE 4865 02:58:49,546 --> 02:58:50,514 DISCUSSED AND BIOMARKERS AND 4866 02:58:50,514 --> 02:58:51,882 IMAGING AND I WILL TALK ABOUT 4867 02:58:51,882 --> 02:59:02,292 THAT IN THE NEXT SLIDES. 4868 02:59:02,993 --> 02:59:03,927 NEXT SLIDE. 4869 02:59:03,927 --> 02:59:06,563 SO THE GOOD NEWS IS IN RECENT 4870 02:59:06,563 --> 02:59:08,332 YEARS THERE ARE BEEN REALLY GOOD 4871 02:59:08,332 --> 02:59:11,835 TECHNIQUES ANDDELOPED 4872 02:59:11,835 --> 02:59:16,707 THE DISEASE MORE, 4873 02:59:16,707 --> 02:59:20,110 SO 1 OF THESE SINGLE CELL 4874 02:59:20,110 --> 02:59:21,078 SEQUENCING AND SINGULAR 4875 02:59:21,078 --> 02:59:25,282 NUCLEOTIDES CLER SEQUENCING AND 4876 02:59:25,282 --> 02:59:26,650 SPATIAL TRANSCRIPTOMICS, THIS 4877 02:59:26,650 --> 02:59:28,485 COULD GIVE US DISEASE INSIGHTS, 4878 02:59:28,485 --> 02:59:29,853 NOVEL TARGETS, BI ONY MARKERS 4879 02:59:29,853 --> 02:59:31,488 AND ALSO CLUES OF WHAT WE CAN 4880 02:59:31,488 --> 02:59:36,927 LOOK AT IN THE CT SCANS. 4881 02:59:36,927 --> 02:59:38,562 THERE'S NOW ALGORITHMS AND NEW 4882 02:59:38,562 --> 02:59:42,199 WAYS TO LOOK AT THE HRCT SCANS 4883 02:59:42,199 --> 02:59:45,068 AND THERE ARE ALSO COHORT FOR 4884 02:59:45,068 --> 02:59:48,172 FIRST DEGREE RELATIVES THAT HAVE 4885 02:59:48,172 --> 02:59:49,439 BEEN FOLLOWED FOR A PERIOD OF 4886 02:59:49,439 --> 02:59:51,108 TIME THAT CAN GIVE US INSIGHT 4887 02:59:51,108 --> 02:59:55,479 INTO EARLIER DEC. 4888 02:59:55,479 --> 02:59:56,280 SO NEXT SLIDE. 4889 02:59:56,280 --> 03:00:00,584 SO I THINK 1 OF THE KEY ADVANCES 4890 03:00:00,584 --> 03:00:02,119 IN THIS FIELD, AND 3 LEGS 4891 03:00:02,119 --> 03:00:06,356 WAS THE DEVELOPMENT OF THE IPFT 4892 03:00:06,356 --> 03:00:07,891 CELL ATLAS WHICH IS A 4893 03:00:07,891 --> 03:00:12,496 COMBINATION OF SINGLE CELL DATA 4894 03:00:12,496 --> 03:00:14,264 FROM X-PLANTED IPF LUNGS FROM 4895 03:00:14,264 --> 03:00:23,407 SEVERAL LABS AROUND THE CO 4896 03:00:23,407 --> 03:00:24,575 INCLUDING NAFTALI KAMINSKI'S 4897 03:00:24,575 --> 03:00:26,910 LAB, SO WE'VE TALKED TO 4898 03:00:26,910 --> 03:00:28,278 INDUSTRY, EXTENSIVELY USES THIS. 4899 03:00:28,278 --> 03:00:30,347 IT'S BEEN USED TO NOT ONLY 4900 03:00:30,347 --> 03:00:33,884 UNDERSTAND THE BIOLOGY, BUT ALSO 4901 03:00:33,884 --> 03:00:35,052 VALIDATE DRUG TARGETS, IT CAN 4902 03:00:35,052 --> 03:00:37,254 ALSO BE USED TO LOOK FOR 4903 03:00:37,254 --> 03:00:39,623 BIOMARKERS, SO IT'S BEEN HIGHLY, 4904 03:00:39,623 --> 03:00:40,123 HIGHLY USED. 4905 03:00:40,123 --> 03:00:41,625 THE DATA I HAVE HERE FOR THE 4906 03:00:41,625 --> 03:00:43,026 NUMBER OF USERS IS ACTUALLY A 4907 03:00:43,026 --> 03:00:44,895 LITTLE BIT OLD, SO IT'S PROBABLY 4908 03:00:44,895 --> 03:00:45,696 MUCH HIGHER THAN THAT. 4909 03:00:45,696 --> 03:00:47,231 AND THERE HAVE BEEN KEY 4910 03:00:47,231 --> 03:00:50,934 FINDINGS, LIKE THE PRESENCE OF 4911 03:00:50,934 --> 03:00:54,271 THESEANT BASALOID CELLS, 4912 03:00:54,271 --> 03:00:56,907 TO PROINFLAMMATORY FIBROBLASTS 4913 03:00:56,907 --> 03:00:58,375 AND WE'VE ALSO SPONSORED SOME 4914 03:00:58,375 --> 03:01:03,347 RESEARCH MORE RECENTLY IN THE 4915 03:01:03,347 --> 03:01:05,849 KAMINSKI LAB, TO LOOK AT 4916 03:01:05,849 --> 03:01:07,417 PREMATURE AND MATURE LESIONS, SO 4917 03:01:07,417 --> 03:01:08,952 INSTEAD OF TAKING THE WHOLE LUNG 4918 03:01:08,952 --> 03:01:11,255 AND PARSING OUT THE CELL, 4919 03:01:11,255 --> 03:01:14,091 LOOKING AT THE CELLS AND IF 4920 03:01:14,091 --> 03:01:15,959 THEY'RE DIFFERENT WHEN THEY'RE 4921 03:01:15,959 --> 03:01:18,395 FULLY MATURE AND PHOBE ROTTIC. 4922 03:01:18,395 --> 03:01:22,532 AND THEY ALSO DEVELOPED THE IPF 4923 03:01:22,532 --> 03:01:24,635 DRUG CONNECT OHM, WHERE YOU CAN 4924 03:01:24,635 --> 03:01:29,973 CONNECK THEECS FROM THE 4925 03:01:29,973 --> 03:01:31,508 CELL ATLAS SOME REPURPOSED DRUGS 4926 03:01:31,508 --> 03:01:33,043 SO CAN YOU MATCH THEM UP AND 4927 03:01:33,043 --> 03:01:35,445 FIND DRUGS THAT MIGHT WORK FOR 4928 03:01:35,445 --> 03:01:36,280 PATIENTS WITH PULMONARY 4929 03:01:36,280 --> 03:01:38,248 FIBROSIS, AND THEN WE ALSO 4930 03:01:38,248 --> 03:01:40,417 SPONSORED WORK WITH SINGLE CELL 4931 03:01:40,417 --> 03:01:42,586 DATA ON PRECISION CUT LUNG 4932 03:01:42,586 --> 03:01:44,454 SLICES THAT HAVE BEEN INDUCED 4933 03:01:44,454 --> 03:01:46,957 WITH A FIBROTIC COCKTAIL, TO SEE 4934 03:01:46,957 --> 03:01:53,563 HOW THIS MATCHES UP WITH THENHER 4935 03:01:53,563 --> 03:01:54,731 STUDYING MORE COMPOUNDS FOR DRUG 4936 03:01:54,731 --> 03:01:59,469 DEVELOPMENT AND THIS HAS BEEN IN 4937 03:01:59,469 --> 03:02:06,009 COLLABORATION WITH MELANIE KONI 4938 03:02:06,009 --> 03:02:06,710 GHOFF, AND OTHERS. 4939 03:02:06,710 --> 03:02:07,044 NEXT SLIDE. 4940 03:02:07,044 --> 03:02:11,548 SO 1 THING THAT WE'RE REALLY 4941 03:02:11,548 --> 03:02:13,317 INTERESTED IN IS SHORING UP SOME 4942 03:02:13,317 --> 03:02:16,119 OF THESE RESOURCES, TRYING AS A 4943 03:02:16,119 --> 03:02:17,054 FOUNDATION, CAN REALLY HELP MAKE 4944 03:02:17,054 --> 03:02:18,088 SOME OF THESE RESOURCES 4945 03:02:18,088 --> 03:02:19,056 AVAILABLE FOR DRUG DEVELOPMENT 4946 03:02:19,056 --> 03:02:21,625 AND WE FEEL LIKE WE'RE IN A 4947 03:02:21,625 --> 03:02:22,392 UNIQUE POSITION. 4948 03:02:22,392 --> 03:02:24,494 WE HAVE FUNDED ACADEMIC LABS, 4949 03:02:24,494 --> 03:02:27,831 AND I HAVE A BACKGROUND IN DRUG 4950 03:02:27,831 --> 03:02:28,565 DEVELOPMENT FROM INDUSTRY WHERE 4951 03:02:28,565 --> 03:02:30,734 THERE'S A GAP THERE, WE'VE HEARD 4952 03:02:30,734 --> 03:02:34,404 ABOUT THE VALLEY OF DEATH WHERE 4953 03:02:34,404 --> 03:02:36,807 ACADEMIC HAS GREAT IDEAS, 4954 03:02:36,807 --> 03:02:38,508 INDUSTRY GREAT DRUG DEVELOPERS, 4955 03:02:38,508 --> 03:02:40,510 BUT THERE'S A GAP, THERE'S AN IN 4956 03:02:40,510 --> 03:02:42,045 BETWEEN AND HOW CAN WE ADDRESS 4957 03:02:42,045 --> 03:02:45,315 THIS IN AND 1 THING WE NEED NEED 4958 03:02:45,315 --> 03:02:46,283 BETTER TOOLS, SO INDUSTRY 4959 03:02:46,283 --> 03:02:47,818 DOESN'T ALWAYS HAVE ACCESS TO 4960 03:02:47,818 --> 03:02:49,252 THE HUMAN TISSUE. 4961 03:02:49,252 --> 03:02:50,787 YOU KNOW, SOME DO BUT WE'VE BEEN 4962 03:02:50,787 --> 03:02:52,255 WORKING WITH INDUSTRY TO 4963 03:02:52,255 --> 03:02:53,757 UNDERSTAND THIS GAP, AND 1 THING 4964 03:02:53,757 --> 03:02:57,861 IS, IF WE CAN PROVIDE CRYO 4965 03:02:57,861 --> 03:02:59,696 PRESERVED TISSUE OR REFRIGERATED 4966 03:02:59,696 --> 03:03:02,733 TISSUE, THEN WE CAN CREATE A 4967 03:03:02,733 --> 03:03:04,801 SOURCE OF THE TISSUE FOR DRUG 4968 03:03:04,801 --> 03:03:05,068 TESTING. 4969 03:03:05,068 --> 03:03:09,072 SO WE'VE BEEN WORKING WITH 4970 03:03:09,072 --> 03:03:12,576 INDUSTRY TO DEVELOP A CONSORTIUM 4971 03:03:12,576 --> 03:03:15,312 WHERE WE WOULD CO-FUND SOME 4972 03:03:15,312 --> 03:03:18,515 ASSAYS TO NUMBER 1 VALIDATE THIS 4973 03:03:18,515 --> 03:03:19,683 CAN BE DONE, SO RIGHT NOW YOU 4974 03:03:19,683 --> 03:03:21,318 CAN BUY THE FROZEN TISSUE BUT 4975 03:03:21,318 --> 03:03:22,819 OFTEN YOU DON'T KNOW WHERE IT'S 4976 03:03:22,819 --> 03:03:24,421 COMING FROM OR ANYTHING ABOUT 4977 03:03:24,421 --> 03:03:27,624 THE PATIENT THAT IT CAME FROM, 4978 03:03:27,624 --> 03:03:28,925 YOU KNOW, SO, YOU DON'T KNOW A 4979 03:03:28,925 --> 03:03:33,597 LOT ABOUT IT AND EXACTLY WHAT IT 4980 03:03:33,597 --> 03:03:33,897 REPRESENTS. 4981 03:03:33,897 --> 03:03:44,207 SO THE NEXT SLIDE. 4982 03:03:45,342 --> 03:03:48,311 SO SOME STUDIES HAVE BEEN DONE 4983 03:03:48,311 --> 03:03:50,714 TO SHOW THIS IS POSSIBLE IN 4984 03:03:50,714 --> 03:03:50,947 PUBLIC. 4985 03:03:50,947 --> 03:03:54,184 SO A STUDY WITTU THE CRYO 4986 03:03:54,184 --> 03:03:55,552 PRESERVING CUT SLICES SHOWSHE 4987 03:03:55,552 --> 03:03:58,455 VIABILITY AFTER 7 AND 14 DAYS SO 4988 03:03:58,455 --> 03:03:59,756 IT WAS PROPRIETARY METHOD OF 4989 03:03:59,756 --> 03:04:02,759 FREEZING THE TISSUE AND THEN 4990 03:04:02,759 --> 03:04:04,061 AFTER THAWING IT AT 7 AND 14 4991 03:04:04,061 --> 03:04:05,829 DAYS IT WAS COMPARED TO NEVER 4992 03:04:05,829 --> 03:04:07,998 FROZEN TISSUE, SO THE VIABILITY 4993 03:04:07,998 --> 03:04:09,099 AND PROTEIN CONTENT LOOKED 4994 03:04:09,099 --> 03:04:09,533 PRETTY GOOD. 4995 03:04:09,533 --> 03:04:19,743 SO THE NEXT SLIDE. 4996 03:04:19,743 --> 03:04:21,745 AND THEN THE SECRETION OF 4997 03:04:21,745 --> 03:04:25,816 CERTAIN CYTOKINES AND SOME OF 4998 03:04:25,816 --> 03:04:28,085 THESE WERE PRESERVED OVER TIME 4999 03:04:28,085 --> 03:04:29,386 AND SOME DID NOT PRESERVE AT ALL 5000 03:04:29,386 --> 03:04:31,121 AND I THINK THAT'S IMPORTANT. 5001 03:04:31,121 --> 03:04:35,225 WE'RE NOT GOING TO GET A PERFECT 5002 03:04:35,225 --> 03:04:36,593 REPLICA OF LIVING HUMAN LUNG, 5003 03:04:36,593 --> 03:04:38,795 BUT WHAT IS IMPORTANT IS TO KNOW 5004 03:04:38,795 --> 03:04:41,798 WHAT THE TISSUE DOES REPRESENT 5005 03:04:41,798 --> 03:04:43,900 AND WHAT CAN BE REPRODUCED. 5006 03:04:43,900 --> 03:04:45,402 N YOU WOULD KNOW WHAT THE 5007 03:04:45,402 --> 03:04:46,803 TISSUE IS USEFUL FOR, SO 5008 03:04:46,803 --> 03:04:48,538 INDUSTRY COULD LOOK AND SEE 5009 03:04:48,538 --> 03:04:50,040 WHETHER THEIR TARGET IS THERE, 5010 03:04:50,040 --> 03:04:51,541 AND WHETHER THEY CAN ACTUALLY 5011 03:04:51,541 --> 03:05:01,785 USE THIS FROZEN TISSUE. 5012 03:05:01,785 --> 03:05:02,452 SO NEXT SLIDE. 5013 03:05:02,452 --> 03:05:04,654 THIS SLIDE JUST SHOWS THE 5014 03:05:04,654 --> 03:05:08,625 CELLULAR DIVERSITY THAT'S 5015 03:05:08,625 --> 03:05:10,293 MAINTAIN AND AGAIN SOME CELL 5016 03:05:10,293 --> 03:05:12,929 TYPES SUCH AS AT2 ARE LOWER IN 5017 03:05:12,929 --> 03:05:15,565 ABUNDANCE AFTER YOU FREEZE AND 5018 03:05:15,565 --> 03:05:18,135 THAW THE TISSUE, WRASSE OTHERS 5019 03:05:18,135 --> 03:05:19,903 LIKE FIBROBLASTS ARE A BIT 5020 03:05:19,903 --> 03:05:28,478 OVERREPRESENTED. NEXT SLIDE. 5021 03:05:28,478 --> 03:05:31,581 NOW, WORKING WITH 5022 03:05:31,581 --> 03:05:33,517 INDUSTRY PARTNERS, THE STUDY 5023 03:05:33,517 --> 03:05:37,053 PLAN IS TO GET TISSUE FROM 3 5024 03:05:37,053 --> 03:05:38,889 FIBROTIC LUNGS, WITH MULTIPLE 5025 03:05:38,889 --> 03:05:41,324 SECTIONS TO ASSESS THE 5026 03:05:41,324 --> 03:05:42,492 VIABILITY, PROFIBROTIC 5027 03:05:42,492 --> 03:05:43,193 SECRETION, CELLULAR COMPOSITION, 5028 03:05:43,193 --> 03:05:45,262 AND ALSO TO LOOK AT THE -- 5029 03:05:45,262 --> 03:05:47,898 WHAT'S ALSO IMPORTANT AND AGAIN 5030 03:05:47,898 --> 03:05:48,965 TO BRIDGE THE GAP BETWEEN 5031 03:05:48,965 --> 03:05:52,569 ACADEMIC AND INDUSTRY IS HOW DO 5032 03:05:52,569 --> 03:05:55,005 THESE DRUGS ACT IN THESE MODELS. 5033 03:05:55,005 --> 03:05:57,073 SO IT'S IMPORTANT TO RUN DOSE 5034 03:05:57,073 --> 03:05:59,009 RESPONSE CURVES AND REALLY 5035 03:05:59,009 --> 03:05:59,376 UNDERSTAND. 5036 03:05:59,376 --> 03:06:08,552 SO ESPECIALLY STANDARD OF CARE, 5037 03:06:08,552 --> 03:06:11,555 SO NINTEDANIB, AND THE ALK5 5038 03:06:11,555 --> 03:06:13,790 INHIBITOR AND THEN ROUGH ATOM 5039 03:06:13,790 --> 03:06:14,758 FIBROTIC COCKTAIL ISSUES AS 5040 03:06:14,758 --> 03:06:15,492 WELL. 5041 03:06:15,492 --> 03:06:16,726 WE WILL CAREFULLY COLLECT THEM 5042 03:06:16,726 --> 03:06:19,062 SO WE KNOW FROM WHAT PART OF THE 5043 03:06:19,062 --> 03:06:21,031 F THEY ARE FROM AND HOW 5044 03:06:21,031 --> 03:06:26,603 FIBROTIC THEY ARE. 5045 03:06:26,603 --> 03:06:27,804 SO NEXT SLIDE. 5046 03:06:27,804 --> 03:06:30,207 SO ANOTH THAT 3 LAKES IS 5047 03:06:30,207 --> 03:06:33,276 VERY INTERESTED IN IS 5048 03:06:33,276 --> 03:06:34,044 UNDERSTANDING EARLY DISEASE. 5049 03:06:34,044 --> 03:06:37,013 SO THE EARLIER WE CAN DIAGNOSE, 5050 03:06:37,013 --> 03:06:39,216 THIS JUST SHOWS THAT BY THE TIME 5051 03:06:39,216 --> 03:06:40,984 YOU'RE DIAGNOSED, YOU'RE PRETTY 5052 03:06:40,984 --> 03:06:42,586 FIBROTIC SO IF WE COULD DIAGNOSE 5053 03:06:42,586 --> 03:06:43,920 EARLIER AND TREAT EARLIER, 5054 03:06:43,920 --> 03:06:44,854 THAT'S THE ONLY WAY WE'RE GOING 5055 03:06:44,854 --> 03:06:46,723 TO GET TO A CURE, SO HOW ARE WE 5056 03:06:46,723 --> 03:06:47,891 GOING TO DO THAT? 5057 03:06:47,891 --> 03:06:52,495 WHEN THE END POINT RIGHT NOW IS 5058 03:06:52,495 --> 03:06:54,264 FVC, SO WE NEED BETTER 5059 03:06:54,264 --> 03:06:55,365 BIOMARKERS AND END POINTS TO DO 5060 03:06:55,365 --> 03:06:55,732 THIS. 5061 03:06:55,732 --> 03:06:58,134 SO 2 THINGS WE NEED TO 5062 03:06:58,134 --> 03:07:00,103 UNDERSTAND THE DISEASE AND 5063 03:07:00,103 --> 03:07:01,137 DEVELOP BIOMARKERS AND END 5064 03:07:01,137 --> 03:07:04,808 POINTS. 5065 03:07:04,808 --> 03:07:05,542 SO NEXT SLIDE. 5066 03:07:05,542 --> 03:07:09,145 SO WE'VE BEEN WORKING WITH JOHN 5067 03:07:09,145 --> 03:07:10,180 [INDISCERNIBLE] AND NICK 5068 03:07:10,180 --> 03:07:13,917 [INDISCERNIBLE] IN A STUDY WE 5069 03:07:13,917 --> 03:07:17,587 CALL 1 POINT FO, THEY HAVE CT 5070 03:07:17,587 --> 03:07:18,822 SCANS FROM OVER 10 YEARS AGO, 5071 03:07:18,822 --> 03:07:20,323 SOME ARE 12 TO 14 YEARS AGO, SO 5072 03:07:20,323 --> 03:07:21,958 IF YOU LOOK AT THE LEFT, THERE'S 5073 03:07:21,958 --> 03:07:24,494 2 LINES THAT START OUT, SO THEY 5074 03:07:24,494 --> 03:07:27,464 EITHER HAD ILAs OR THEY HAD NO 5075 03:07:27,464 --> 03:07:27,897 FINDINGS WHATSOEVER. 5076 03:07:27,897 --> 03:07:29,466 AND AS IT GOES TO THE RIGHT WHEN 5077 03:07:29,466 --> 03:07:32,335 IT DROPS DOWN, SOME OF THESE 5078 03:07:32,335 --> 03:07:33,069 PEOPLE HAVE DIRECTINGED, THEIR 5079 03:07:33,069 --> 03:07:34,371 DISEASE HAVE PROGRESSED AND SOME 5080 03:07:34,371 --> 03:07:35,972 OF THEMHE PULMONARYS, 5081 03:07:35,972 --> 03:07:37,607 SO WE CAN TAKE THESE AND PUT 5082 03:07:37,607 --> 03:07:39,676 THEM INTO BUCKETS BECAUSE WE CAN 5083 03:07:39,676 --> 03:07:41,845 LOOK BACK, SO WE SELECTED0 5084 03:07:41,845 --> 03:07:43,613 SAMPLES FROM EACH GROUP AND DID 5085 03:07:43,613 --> 03:07:46,016 SINGLE CELL ANALYSIS AND HERE WE 5086 03:07:46,016 --> 03:07:48,451 SHOW REPRESENTATIVE WORK ON AT2 5087 03:07:48,451 --> 03:07:50,153 MOLECULES AND YOU CAN JUST SEE 5088 03:07:50,153 --> 03:07:51,021 THEY'RE DIFFERENT. 5089 03:07:51,021 --> 03:07:52,822 SO WHAT THIS SHOW SYSTEM THAT 5090 03:07:52,822 --> 03:07:54,958 THERE ARE MOLECULAR DIFFERENCES 5091 03:07:54,958 --> 03:07:56,593 VERY EARLY ON THAT PROCEED WHAT 5092 03:07:56,593 --> 03:07:59,396 WE CAN PICK UP IN THE CT SCANS. 5093 03:07:59,396 --> 03:08:01,131 SO IF WE CAN FIND BIOMARKERS 5094 03:08:01,131 --> 03:08:03,366 THAT WE CAN THEN DETECT IN THE 5095 03:08:03,366 --> 03:08:05,402 BLOOD, MY DREAM WOULD BE YOU GO 5096 03:08:05,402 --> 03:08:07,137 INTO THE DOCTOR, YOU A BLOOD 5097 03:08:07,137 --> 03:08:08,672 TEST, IF YOU HAVE A FAMILY 5098 03:08:08,672 --> 03:08:10,440 MEMBER, YOU'RE AT RISK, THEN YOU 5099 03:08:10,440 --> 03:08:12,542 GO GET A CT SCAN SO YOU CAN GET 5100 03:08:12,542 --> 03:08:13,810 DIAGNOSED MUCH EARLIER AND IF WE 5101 03:08:13,810 --> 03:08:16,246 UNDERSTAND THE BIOLOGY OF WHAT'E 5102 03:08:16,246 --> 03:08:19,015 DRUGGINGS OUT THERE THAT -- 5103 03:08:19,015 --> 03:08:21,017 DRUGS THAT CAN ACTUALLY TREAT 5104 03:08:21,017 --> 03:08:27,791 EARLIER. 5105 03:08:27,791 --> 03:08:28,591 NEXT SLIDE. 5106 03:08:28,591 --> 03:08:30,560 SO THERE HAS BEEN SOME 5107 03:08:30,560 --> 03:08:31,661 BIOMARKERS, WE STILL HAVE A LONG 5108 03:08:31,661 --> 03:08:32,595 WAY TO GO. 5109 03:08:32,595 --> 03:08:34,698 THESE ARE THE BIOMARKERS THAT 5110 03:08:34,698 --> 03:08:36,766 HAVE BEEN IDENTIFIED, I WON'T GO 5111 03:08:36,766 --> 03:08:40,503 THROUGH THEM ALL, SOME OF THEM 5112 03:08:40,503 --> 03:08:42,572 ARE FIBROTIC DECS, DIAGNOSTIC, 5113 03:08:42,572 --> 03:08:43,873 PROGNOSTIC THAT PREDICT THE 5114 03:08:43,873 --> 03:08:45,508 OUTCOME AND SOME IN RESPONSE TO 5115 03:08:45,508 --> 03:08:48,445 THERAPY BUT NONE OF THESE HAVE 5116 03:08:48,445 --> 03:08:49,346 BEEN APPROVED FOR CLINICAL 5117 03:08:49,346 --> 03:08:51,081 TRIALS AND AGAIN AS DISCUSSED 5118 03:08:51,081 --> 03:08:53,016 EARLIER, WE PROBABLY NEED A 5119 03:08:53,016 --> 03:08:54,384 PANEL OF BIOMARKERS WITH 5120 03:08:54,384 --> 03:08:57,387 BIOMARKERS ALONE WILL DO THE 5121 03:08:57,387 --> 03:09:00,757 JOB. 5122 03:09:00,757 --> 03:09:01,191 NEXT SLIDE. 5123 03:09:01,191 --> 03:09:05,028 SO THERE ARE SOME STUDIES ON 5124 03:09:05,028 --> 03:09:07,530 COMBINATIONS, 1 OF THE FIRST 5125 03:09:07,530 --> 03:09:12,936 STUDIES WAS FROM IVAN ROSAS IN 5126 03:09:12,936 --> 03:09:16,573 COLLEAGUES IN 2008 WITH MMP 7, 5127 03:09:16,573 --> 03:09:22,812 MPP1 AND MMP8, AND IGFP1 AND 5128 03:09:22,812 --> 03:09:23,747 TGF-RECEPTOR AND YOU CAN SEE 5129 03:09:23,747 --> 03:09:25,148 THAT SOME OF THESE HAVE BEEN 5130 03:09:25,148 --> 03:09:26,216 REPEATED OVER AND OVER. 5131 03:09:26,216 --> 03:09:28,184 THERE'S HAVE BEEN A COUPLE OTHER 5132 03:09:28,184 --> 03:09:29,953 STUDIES BUT AGAIN, NOTHING YET 5133 03:09:29,953 --> 03:09:32,055 HAS MADE IT TO THE POINT THAT IT 5134 03:09:32,055 --> 03:09:34,124 HAS BEEN REPRODUCED AND CAN BE 5135 03:09:34,124 --> 03:09:35,892 USED AS A CLINICAL EITHER 5136 03:09:35,892 --> 03:09:37,727 COMBINATION OF BIOMARKER AND TO 5137 03:09:37,727 --> 03:09:40,330 DO THIS WE'RE GOING TO NEED TO 5138 03:09:40,330 --> 03:09:42,365 VALIDATE IT IN MULTIPLE COHORTS 5139 03:09:42,365 --> 03:09:43,767 OF PATIENTS. 5140 03:09:43,767 --> 03:09:49,172 SO THE NEXT SLIDE. 5141 03:09:49,172 --> 03:09:51,608 SO THERE'S A PROLIFIC CONSORTIUM 5142 03:09:51,608 --> 03:09:52,542 WHICH IS EXACTLY WHAT THEY'RE 5143 03:09:52,542 --> 03:09:54,310 TRYING TO DO, IT'S A NUMBER OF 5144 03:09:54,310 --> 03:09:55,845 INDUSTRY MEMBERS AND 3 LAKES IS 5145 03:09:55,845 --> 03:09:58,081 PARTICIPATING IN THIS AS WELL AS 5146 03:09:58,081 --> 03:09:59,783 THE PULMONARY FIBROSIS 5147 03:09:59,783 --> 03:10:01,151 FOUNDATION, SO THEY IDENTIFY 12 5148 03:10:01,151 --> 03:10:03,420 BIOMARKERS FROM THE LITERATURE 5149 03:10:03,420 --> 03:10:05,054 AND REALLY VALIDATED ASSAYS AND 5150 03:10:05,054 --> 03:10:06,723 THEN LOOKED FOR THEM AND 5151 03:10:06,723 --> 03:10:11,094 COMPARED THEM TO PREDICTED FVC 5152 03:10:11,094 --> 03:10:12,228 TRANSPLANT-FREE SURVIVAL AND 5153 03:10:12,228 --> 03:10:12,962 DEATH AND LUNG TRANSPLANT AND 5154 03:10:12,962 --> 03:10:15,965 THE 1 THAT ROSE TO THE TOP IS 5155 03:10:15,965 --> 03:10:17,734 SPD AND THERE'S OTHERS 1S BUT 5156 03:10:17,734 --> 03:10:20,370 E NOT L AT A COUPLE 5157 03:10:20,370 --> 03:10:21,905 OTHER COHORTS TO TRY TO 5158 03:10:21,905 --> 03:10:23,206 REPRODUCE THIS DATA BECAUSE 5159 03:10:23,206 --> 03:10:24,674 THEY'RE REALLY TRYING TO FIND 5160 03:10:24,674 --> 03:10:26,509 SOMETHING THAT THEY CAN THEN USE 5161 03:10:26,509 --> 03:10:28,344 AS A VALIDATED END POINT IN 5162 03:10:28,344 --> 03:10:30,079 CLINICAL TRIALS. 5163 03:10:30,079 --> 03:10:32,282 NEXT STUDY. 5164 03:10:32,282 --> 03:10:32,849 NEXT SLIDE, SORRY. 5165 03:10:32,849 --> 03:10:34,784 SO WE NEED MORE VALIDATED 5166 03:10:34,784 --> 03:10:37,020 BIOMARKERS FOR BOTH EARLY AND 5167 03:10:37,020 --> 03:10:39,189 ESTABLISHED DISEASE SINCE FVC IS 5168 03:10:39,189 --> 03:10:40,056 THE ONLY APPROVED BIOMARKER AND 5169 03:10:40,056 --> 03:10:43,226 IF WE WANT TO TREAT EARLIER, 5170 03:10:43,226 --> 03:10:45,228 THERE WON'T BE ANY CHANGES IN 5171 03:10:45,228 --> 03:10:47,831 FVC SO WE HAVE TO COME UP WITH 5172 03:10:47,831 --> 03:10:48,298 SOMETHING. 5173 03:10:48,298 --> 03:10:52,001 HRCT SCANS, WE'VE HEARD ABOUT 5174 03:10:52,001 --> 03:10:53,770 THAT AND THOSE ARE LINKED TO 5175 03:10:53,770 --> 03:10:55,271 PROGNOSIS BUT YET HAVE NOTN 5176 03:10:55,271 --> 03:10:55,738 VALIDATED YET. 5177 03:10:55,738 --> 03:10:57,273 THERE'S WORK TO DO THAT BUT WE 5178 03:10:57,273 --> 03:10:59,275 HAVE A WAYS TO GO. 5179 03:10:59,275 --> 03:11:00,777 SINGLE BIOMARKERS MAY NOT BE 5180 03:11:00,777 --> 03:11:02,879 SUFFICIENT, WE MAY NEED A 5181 03:11:02,879 --> 03:11:03,913 COMPOSITE BIOMARKER AND A LOT 5182 03:11:03,913 --> 03:11:05,648 MORE WORK NEEDS TO BE DONE IN 5183 03:11:05,648 --> 03:11:06,816 THIS AREA AND THERE ARE OTHER 5184 03:11:06,816 --> 03:11:09,752 THINGS THAT COULD POSSIBLY USE 5185 03:11:09,752 --> 03:11:12,355 AS BIOMARKERS SUCH AS BREATH, 5186 03:11:12,355 --> 03:11:15,758 VOLATILE, ORGANIC COMPOUNDS OR 5187 03:11:15,758 --> 03:11:17,660 EXOSOMES. 5188 03:11:17,660 --> 03:11:18,695 NEXT SLIDE. 5189 03:11:18,695 --> 03:11:20,330 SO I JUST WANTED TO END WITH A 5190 03:11:20,330 --> 03:11:22,131 FIGURE THAT CAME OUT OF THE 5191 03:11:22,131 --> 03:11:25,101 PROCEEDINGS FROM THE NHLBI 5192 03:11:25,101 --> 03:11:28,538 WORKSHOP THAT WAS HELD ON 5193 03:11:28,538 --> 03:11:29,739 PULMONARY FIBROSIS IN 2022 WHICH 5194 03:11:29,739 --> 03:11:31,040 REALLY SUMMARIZES THE GAPS IN 5195 03:11:31,040 --> 03:11:34,244 THE FIELD AND WHERE WE NEED TO 5196 03:11:34,244 --> 03:11:34,410 GO. 5197 03:11:34,410 --> 03:11:35,778 AND THAT'S -- YOU KNOW THE 5198 03:11:35,778 --> 03:11:37,580 MODELS THAT WE'VE HEARD ABOUT 5199 03:11:37,580 --> 03:11:41,551 UNDERSTANDING EARLY DEC AND RISK 5200 03:11:41,551 --> 03:11:49,158 FACTORS, CLINICAL TRIALS AND 5201 03:11:49,158 --> 03:11:49,926 BIOMARKERS. 5202 03:11:49,926 --> 03:11:52,262 THREE LAKES IS PROUD OF THE 5203 03:11:52,262 --> 03:11:53,530 DIFFERENCE THEY'VE MADE IN THIS 5204 03:11:53,530 --> 03:11:55,131 FIELD AS A PRIVATE FOUNDATION 5205 03:11:55,131 --> 03:11:56,699 AND AS A PRIVATE FOUNDATION 5206 03:11:56,699 --> 03:11:58,001 WE'VE NOT HAD OTHER PEOPLE YOIN 5207 03:11:58,001 --> 03:11:59,202 US IN THIS MISSION SO I WANTED 5208 03:11:59,202 --> 03:12:01,838 TO LET PEOPLE KNOW THAT IN 2025, 5209 03:12:01,838 --> 03:12:03,473 WE'RE PLANNING TO CHANGE INTO A 5210 03:12:03,473 --> 03:12:05,141 PUBLIC FOUNDATION SO HOPEFULLY 5211 03:12:05,141 --> 03:12:06,276 OTHERS WILL JOIN US ON THIS 5212 03:12:06,276 --> 03:12:08,344 MISSION AND WE CAN CONTINUE THIS 5213 03:12:08,344 --> 03:12:13,416 WORK. . 5214 03:12:13,416 --> 03:12:14,551 THANK YOU. 5215 03:12:14,551 --> 03:12:15,118 >> THANKS SHERYL, LOOKING 5216 03:12:15,118 --> 03:12:17,253 FORWARD TO TALKING TO YOU DURING 5217 03:12:17,253 --> 03:12:18,388 THE PANEL DISCUSSION, WE WILL 5218 03:12:18,388 --> 03:12:20,757 MOVE INTO THE LAST SPEAKER OF 5219 03:12:20,757 --> 03:12:28,531 THIS SESSION, DR. Mc CORMAC K. 5220 03:12:28,531 --> 03:12:29,065 >> OKAY, THANK YOU. 5221 03:12:29,065 --> 03:12:32,168 I WOULD LIKE TO THANK ALL THE 5222 03:12:32,168 --> 03:12:33,002 ORGANIZERS FOR THE OPPORTUNITY 5223 03:12:33,002 --> 03:12:38,942 TO TELL YOU ABOUT PROGRESS IN 5224 03:12:38,942 --> 03:12:41,344 LAM, AND THIS IS THE STORY OF A 5225 03:12:41,344 --> 03:12:42,579 GRASS ROOTS EFFORT LAUNCHED BY A 5226 03:12:42,579 --> 03:12:44,314 MOTHER FOR THE BENEFIT OF HER 5227 03:12:44,314 --> 03:12:47,083 DAUGHTER, NOT UNLIKE THE STORY 5228 03:12:47,083 --> 03:12:48,217 MICHELLE MANNION WAS TELLING 5229 03:12:48,217 --> 03:12:50,420 WHICH LED TO AN EFFECTIVE 5230 03:12:50,420 --> 03:12:57,126 THERAPY. 5231 03:12:57,126 --> 03:12:58,061 NEXT SLIDE, PLEASE. 5232 03:12:58,061 --> 03:12:59,562 MY CONFLICTS THAT ARE RELEVANT 5233 03:12:59,562 --> 03:13:01,564 TO TODAY'S TALK INCLUDE A 5234 03:13:01,564 --> 03:13:02,498 FEDERAL AND UNRESTRICTED 5235 03:13:02,498 --> 03:13:07,070 INDUSTRY GRANTS, A PAT EPT FOR A 5236 03:13:07,070 --> 03:13:09,172 DIAGNOSTIC BIOMARKER CALLED 5237 03:13:09,172 --> 03:13:09,906 VEG-FD, ALL ROYALTIES ARE 5238 03:13:09,906 --> 03:13:11,841 DIRECTED TO OUR INSTITUTION AND 5239 03:13:11,841 --> 03:13:19,349 DONATION OF DRUGS BY PHARMA FOR 5240 03:13:19,349 --> 03:13:19,582 TRIALS. 5241 03:13:19,582 --> 03:13:19,983 NEXT SLIDE PLEASE. 5242 03:13:19,983 --> 03:13:22,085 MY FRAME OF REFERENCE IS THAT IA 5243 03:13:22,085 --> 03:13:23,019 WAS THE FOUNDING SCIENTIFIC 5244 03:13:23,019 --> 03:13:24,654 DIRECTOR OF THE LAM FOUNDATION 5245 03:13:24,654 --> 03:13:27,056 AND SERVED IN THAT ROLE FOR 25 5246 03:13:27,056 --> 03:13:29,392 YEARS THROUGH JANUARY OF 2021, 5247 03:13:29,392 --> 03:13:31,094 VOLUNTARILY AND I WAS THE 5248 03:13:31,094 --> 03:13:31,828 PRINCIPAL INVESTIGATOROT TRIAL 5249 03:13:31,828 --> 03:13:34,030 EMPLOY I WAS DISCUSSING THE 5250 03:13:34,030 --> 03:13:36,332 MILES TRIAL, I WILL BE 5251 03:13:36,332 --> 03:13:37,634 DISCUSSING, MY PRIMARY OBJECTIVE 5252 03:13:37,634 --> 03:13:41,604 IS TO HIGHLIGHT THE ROLE OF 5253 03:13:41,604 --> 03:13:43,006 ADVOCACY BY THE LAM FOUNDATION 5254 03:13:43,006 --> 03:13:47,176 AND PROGRESS. 5255 03:13:47,176 --> 03:13:47,877 NEXT SLIDE, PLEASE. 5256 03:13:47,877 --> 03:13:49,212 IT'S IMPORTANT I TELL YOU ABOUT 5257 03:13:49,212 --> 03:13:51,414 THE RARE LUNG DISEASE 5258 03:13:51,414 --> 03:13:52,281 CONSORTIUM, MICHELLE ALSO HAD 5259 03:13:52,281 --> 03:13:53,583 SLIDES ABOUT THIS FOR A PERIOD 5260 03:13:53,583 --> 03:13:55,985 OF ABOUT 15 YEARS, WITH A GAP IN 5261 03:13:55,985 --> 03:13:59,055 THE MITTED, BRUCE AND I WERE 5262 03:13:59,055 --> 03:14:01,257 AWARDED A GRANT FROM NCATS AND 5263 03:14:01,257 --> 03:14:03,326 NHLBI TO ESTABLISH A RARE LUNG 5264 03:14:03,326 --> 03:14:05,828 DEC CLINICAL NETWORK OF ABOUT 75 5265 03:14:05,828 --> 03:14:07,397 CLINICS AROUND THE WORLD THAT 5266 03:14:07,397 --> 03:14:09,365 FOCUS REFERRALS TO 1 OR 2 5267 03:14:09,365 --> 03:14:11,200 ACADEMIC INSTITUTIONS WITHIN 5268 03:14:11,200 --> 03:14:12,802 POPULATION CENTERS FOR THE 5269 03:14:12,802 --> 03:14:15,271 PURPOSE OF NURTURING EXPERTISE, 5270 03:14:15,271 --> 03:14:16,506 FACILITATING EXPERT CLINICAL 5271 03:14:16,506 --> 03:14:17,473 CARE AND CONDUCTING RESEARCH. 5272 03:14:17,473 --> 03:14:19,742 IT WAS THROUGH THIS NETWORK THAT 5273 03:14:19,742 --> 03:14:21,411 MOST OF THEMOAM TRIALS HAVE BEEN 5274 03:14:21,411 --> 03:14:29,619 CONDUCTED AND ALSO TRIALS IN 5275 03:14:29,619 --> 03:14:30,186 [INDISCERNIBLE] AND 5276 03:14:30,186 --> 03:14:30,753 [INDISCERNIBLE]. 5277 03:14:30,753 --> 03:14:32,488 THE NETWORK STARTED ON A 5278 03:14:32,488 --> 03:14:33,656 PLATFORM OF CLINICS WHICH 5279 03:14:33,656 --> 03:14:35,291 EXPANDED THEIR SCOPE OF INTEREST 5280 03:14:35,291 --> 03:14:36,826 TO INCLUDE ABOUT 2 DOZEN OTHER 5281 03:14:36,826 --> 03:14:39,629 RARE LUNG DECS SOME OF WHICH ARE 5282 03:14:39,629 --> 03:14:40,830 REPRESENTED BY PATIENT ADVOCACY 5283 03:14:40,830 --> 03:14:42,832 ORGANIZATION SHOWN IN RED AND IN 5284 03:14:42,832 --> 03:14:47,170 BOLD RED FOR THOSE THAT HAVE 5285 03:14:47,170 --> 03:14:48,171 STRONG PATIENTA ADVOCACY 5286 03:14:48,171 --> 03:14:50,206 ORGANIZATIONOCNI ASSOCIATE WIDE 5287 03:14:50,206 --> 03:14:56,546 THEM. 5288 03:14:56,546 --> 03:14:57,647 NEXT SLIDE PLEASE. 5289 03:14:57,647 --> 03:15:01,884 SO LAM IS AN UNCOMMON AND FATAL 5290 03:15:01,884 --> 03:15:05,254 PROGRESSIVE DEC IN YOUNG WOMEN, 5291 03:15:05,254 --> 03:15:07,557 CHARACTERIZED BY SMOOTH MUSCLE 5292 03:15:07,557 --> 03:15:08,925 CELL INFILTRATION AND CYSTIC 5293 03:15:08,925 --> 03:15:11,894 DESTRUCTION OF LUNG TISSUE, ON 5294 03:15:11,894 --> 03:15:13,196 THE IMAGE ON THE RIGHT 5295 03:15:13,196 --> 03:15:15,231 HIGHLIGHTS THE CYSTS AND IT'S 5296 03:15:15,231 --> 03:15:17,433 EASY TO APPRECIATE HOW 5297 03:15:17,433 --> 03:15:18,101 TREMENDOUSLY DISSTRUCTURALLYIVE 5298 03:15:18,101 --> 03:15:19,769 THIS CAN BE TO LUNG 5299 03:15:19,769 --> 03:15:28,644 ARCHITECTURE. 5300 03:15:28,644 --> 03:15:29,345 NEXT SLIDE, PLEASE. 5301 03:15:29,345 --> 03:15:31,748 SO LAM OCCURS MOSTLY IN WOMEN AT 5302 03:15:31,748 --> 03:15:34,016 AN AVERAGE AGE OF ABOUT 35 YEARS 5303 03:15:34,016 --> 03:15:36,986 WITH A RAINCHL FROM 3-85 IN THE 5304 03:15:36,986 --> 03:15:37,286 LITERATURE. 5305 03:15:37,286 --> 03:15:39,856 THERE ARE MEN WHO ARE AFFECTED 5306 03:15:39,856 --> 03:15:42,492 BUT ONLY A FEW SYMPTOMATIC MEN 5307 03:15:42,492 --> 03:15:47,463 THAT I'VE EVER BECOME AWARE OF. 5308 03:15:47,463 --> 03:15:48,731 IT AFFECTS ABOUT 25 WOMEN PER 5309 03:15:48,731 --> 03:15:49,766 MILLION WOMEN. 5310 03:15:49,766 --> 03:15:51,534 THE RATE OF LUNG FUNCTION 5311 03:15:51,534 --> 03:15:54,070 DECLINE BASED ON FEV1 IS ABOUT 5312 03:15:54,070 --> 03:15:57,707 3-15% PER YEAR IN MOST PATIENTS. 5313 03:15:57,707 --> 03:16:00,343 SUCH THAT WITHIN 10 YEARS OF 5314 03:16:00,343 --> 03:16:02,445 SYMPTOM ONSET, 55% OF THE 5315 03:16:02,445 --> 03:16:03,946 PATIENTS ARE BREATHLESS, 20% 5316 03:16:03,946 --> 03:16:06,682 ONOXIEN AND 10% ARE DECEASED. 5317 03:16:06,682 --> 03:16:07,984 THE MEDIAN SURVIVAL IN EARLY 5318 03:16:07,984 --> 03:16:09,252 STUDIES WAS ESTIMATED TO BE 5319 03:16:09,252 --> 03:16:10,920 ABOUT 8 AND HALF YEARS BUT IT'S 5320 03:16:10,920 --> 03:16:12,221 BECOME CLEAR THROUGH POPULATION 5321 03:16:12,221 --> 03:16:14,724 BASED STUDIES THAT IT'S CLOSER 5322 03:16:14,724 --> 03:16:15,758 TO 25 YEARS. 5323 03:16:15,758 --> 03:16:17,493 THERE IS NO CURE FOR THIS 5324 03:16:17,493 --> 03:16:18,995 DISEASE TO THIS DATE. 5325 03:16:18,995 --> 03:16:22,632 THE IMAGE AT THE TOP WAS A 5326 03:16:22,632 --> 03:16:24,700 FORMERLY HEALTHY PATIENT AND 5327 03:16:24,700 --> 03:16:26,435 DISCOVERED SHE WAS 16 WEEKS 5328 03:16:26,435 --> 03:16:27,970 PREGNANT BY URINE DIP STICK AT 5329 03:16:27,970 --> 03:16:29,705 HOME AND SUFFERED A PNEUMOTHORAX 5330 03:16:29,705 --> 03:16:34,443 THE NEXT DAY AND A KOBT 5331 03:16:34,443 --> 03:16:35,845 RALATERAL PNEUMOTHORAX AND SHE 5332 03:16:35,845 --> 03:16:39,148 WAS HOSPITALIZED FOR HER ENTIRE 5333 03:16:39,148 --> 03:16:39,448 PREGNANCY. 5334 03:16:39,448 --> 03:16:41,017 SHE WENT HOME TO OXYGEN AS YOU 5335 03:16:41,017 --> 03:16:42,919 CAN SEE AND SHE REQUIRED A LUNG 5336 03:16:42,919 --> 03:16:43,986 TRANSPLANT A FEW YEARS LATER AND 5337 03:16:43,986 --> 03:16:46,355 YOU CAN SEE IN THE BOTTOM IMAGE 5338 03:16:46,355 --> 03:16:48,825 SHE'S A BIT CURBINGOID AND HER 5339 03:16:48,825 --> 03:16:51,427 DAUGHTER'S MAYBE 4 OR 5 YEARS 5340 03:16:51,427 --> 03:16:51,861 OLD. 5341 03:16:51,861 --> 03:16:53,529 THAT CHILD IS NOW 35 YEARS OLD, 5342 03:16:53,529 --> 03:16:56,065 THE MOM HAD A SECOND LUNG 5343 03:16:56,065 --> 03:16:57,767 TRANSPLANT, THIS CASE 5344 03:16:57,767 --> 03:17:01,103 DEMONSTRATES THAT LAM CAN RESULT 5345 03:17:01,103 --> 03:17:03,406 IN RESPIRATORY FAILURE 5346 03:17:03,406 --> 03:17:05,174 ACCELERATE BIDE HORMONAL 5347 03:17:05,174 --> 03:17:07,643 INFLUENCES. 5348 03:17:07,643 --> 03:17:08,444 NEXT SLIDE, PLEASE. 5349 03:17:08,444 --> 03:17:10,146 ANDREA BURNS WAS THE INSPIRATION 5350 03:17:10,146 --> 03:17:12,348 FOR THE LAM FOUNDATION. 5351 03:17:12,348 --> 03:17:14,984 SHE IS A YOUNG LADY FROM 5352 03:17:14,984 --> 03:17:15,718 CINCINNATI WHO DEVELOPED CHEST 5353 03:17:15,718 --> 03:17:18,821 PAIN ON A FLIGHT TO DENVER IN 5354 03:17:18,821 --> 03:17:19,488 1984. 5355 03:17:19,488 --> 03:17:21,224 SHE WAS RESUSCITATED ON THE 5356 03:17:21,224 --> 03:17:23,092 TARMAC IN DENVER AND FOUND TO 5357 03:17:23,092 --> 03:17:24,727 HAVE A COLLAPSED LUNG, A CHEST 5358 03:17:24,727 --> 03:17:26,796 TUBE WAS PLACED IN THE COLORADO 5359 03:17:26,796 --> 03:17:29,398 EMERGENCY ROOM AND A CT SCANNED 5360 03:17:29,398 --> 03:17:32,034 DIFFUSE CYSTIC LUNG DISEASE, SHE 5361 03:17:32,034 --> 03:17:34,470 TOLD THE SURGEON SHE HAD HAD A 5362 03:17:34,470 --> 03:17:36,339 RARE KIDNEY TUMOR REMOVED BEFORE 5363 03:17:36,339 --> 03:17:38,241 AND HE WAS ABLE TO TELL HER WHAT 5364 03:17:38,241 --> 03:17:39,642 THE PHYSICIANS IN CINCINNATI HAD 5365 03:17:39,642 --> 03:17:41,911 NOT TOLD HER, THAT BASED ON 5366 03:17:41,911 --> 03:17:45,181 THOSE 2 FINDINGS, AND WHERE THE 5367 03:17:45,181 --> 03:17:46,916 KIDNEY TUMOR AND THE CYSTIC 5368 03:17:46,916 --> 03:17:51,420 DISEASE AND THAT SHE HAD LAM AND 5369 03:17:51,420 --> 03:17:53,522 HE RECOMMENDED SHE SEE ME WHEN 5370 03:17:53,522 --> 03:17:55,091 SHE RETURNED TO CINCINNATI AS I 5371 03:17:55,091 --> 03:17:57,226 HAD JUST MOVED TO CINCINNATI. 5372 03:17:57,226 --> 03:17:58,794 NEXT SLIDE ISSUE PLEASE. 5373 03:17:58,794 --> 03:18:03,566 LUCKY FOR HER, HER MOTHER IS SUE 5374 03:18:03,566 --> 03:18:04,734 BURNS WHO ESTABLISHED THE 5375 03:18:04,734 --> 03:18:06,669 FOUNDATION IN HER HONOR. 5376 03:18:06,669 --> 03:18:07,703 SHE MET WITH 9 LAM PATIENTS IN 5377 03:18:07,703 --> 03:18:10,172 HER HOME IN THE EARLY 90S. 5378 03:18:10,172 --> 03:18:11,007 SHE FOWBDED THE FOUNDATION IN 5379 03:18:11,007 --> 03:18:13,075 HER BASEMENT AND ASKED ME TO 5380 03:18:13,075 --> 03:18:14,877 SERVE AS HER VOLUNTEER 5381 03:18:14,877 --> 03:18:15,978 SCIENTIFIC DIRECTOR. 5382 03:18:15,978 --> 03:18:18,047 SHE READ AN ARTICLE SUGGESTING 5383 03:18:18,047 --> 03:18:20,049 CRITICAL NEED FOR REGISTRY BY 5384 03:18:20,049 --> 03:18:23,719 DOCTORS TAYLOR AND RUE AND 5385 03:18:23,719 --> 03:18:23,920 COLBY. 5386 03:18:23,920 --> 03:18:25,321 SHE MARSHALED A LETTER WRITING 5387 03:18:25,321 --> 03:18:27,790 CAMPAIGN TO THE NIH AND DRNG 5388 03:18:27,790 --> 03:18:29,692 INCLUDING PULMONARY PHYSICIANS 5389 03:18:29,692 --> 03:18:31,327 ACROSS THE U.S. 5390 03:18:31,327 --> 03:18:32,962 USING ALL OF OUR STUDENTS AT 5391 03:18:32,962 --> 03:18:34,230 SAINT MICHAELS WHERE SHE TAUGHT 5392 03:18:34,230 --> 03:18:36,098 MUSIC AND HER HUSBAND WAS A 5393 03:18:36,098 --> 03:18:37,700 FOOTBALL COACH AND USING THIS 5394 03:18:37,700 --> 03:18:39,235 TYPE WRITER SHOWN ON THE PANEL 5395 03:18:39,235 --> 03:18:40,569 ON THE BOTTOM. 5396 03:18:40,569 --> 03:18:42,638 SHE TESTIFIED BEFORE CONGRESS 5397 03:18:42,638 --> 03:18:43,439 AND ULTIMATELY [INDISCERNIBLE] 5398 03:18:43,439 --> 03:18:45,775 WHO WAS THEN THE NHLBI DIRECTOR 5399 03:18:45,775 --> 03:18:48,744 INVITED HER TO COME TO THE NIH 5400 03:18:48,744 --> 03:18:50,646 AND TOLD HER HE WOULD COMMISSION 5401 03:18:50,646 --> 03:18:53,015 AN NIH REGISTRY AND AN 5402 03:18:53,015 --> 03:18:54,850 INTRAMURAL LAM PROTOCOL FROM 5403 03:18:54,850 --> 03:18:56,652 THAT POINT FORWARD AND THE 5404 03:18:56,652 --> 03:18:57,687 INTRAMURAL PROTOCOL WAS STILL IN 5405 03:18:57,687 --> 03:19:05,428 PLACE TO THIS DAY. 5406 03:19:05,428 --> 03:19:06,729 NEXT SLIDE, PLEASE. 5407 03:19:06,729 --> 03:19:08,998 SO, SUE AND FRAN FOUNDED THE 5408 03:19:08,998 --> 03:19:11,367 NOWNDATION IN 1995 AND OVER THE 5409 03:19:11,367 --> 03:19:14,837 NEXT 20 YEARS I WAS INVOLVED, 5410 03:19:14,837 --> 03:19:16,439 THEY RAISED 18 MILLION-DOLLAR 5411 03:19:16,439 --> 03:19:17,773 AND DEVOTED 12 MILLION OF THAT 5412 03:19:17,773 --> 03:19:19,842 TO RESEARCH AND FUNDED 155 5413 03:19:19,842 --> 03:19:22,478 RESEARCH GRANTS. 5414 03:19:22,478 --> 03:19:23,646 THEY HELD ANNUAL LAM CONFERENCE 5415 03:19:23,646 --> 03:19:25,581 IN CINCINNATI WHICH WAS FUNDED 5416 03:19:25,581 --> 03:19:30,086 EVERY YEAR BY AN NHLBI R13 GRANT 5417 03:19:30,086 --> 03:19:32,221 WHICH CONVENED ABOUT 150 LAM 5418 03:19:32,221 --> 03:19:34,290 PATIENTS AND ABOUT A HUNDRED 5419 03:19:34,290 --> 03:19:35,458 INVESTIGATORS IN THIS UNIQUE 5420 03:19:35,458 --> 03:19:37,259 ISHT ACTIVE FORUM WHERE THE 5421 03:19:37,259 --> 03:19:38,394 PATIENTS ARE WELCOME IN THE 5422 03:19:38,394 --> 03:19:40,129 SCIENTIFIC SESSIONS AND INVITED 5423 03:19:40,129 --> 03:19:43,032 TO ASK QUESTIONS THERE AND THE 5424 03:19:43,032 --> 03:19:44,000 INVESTIGATORS AND CLINICIANS ARE 5425 03:19:44,000 --> 03:19:45,501 UPDATING THE PATIENTS ON 5426 03:19:45,501 --> 03:19:47,003 SCIENTIFIC ADVANCES ALL ON THE 5427 03:19:47,003 --> 03:19:50,506 SAME SETTING AND IT ENDS WITH A 5428 03:19:50,506 --> 03:19:51,607 FUNDRAISING EVENT THAT USUALLY 5429 03:19:51,607 --> 03:19:52,808 GENERATES SOMEWHERE IN THE 5430 03:19:52,808 --> 03:19:55,778 NEIGHBORHOOD OF 150 TO $200,000 5431 03:19:55,778 --> 03:20:00,950 OR MORE. 5432 03:20:00,950 --> 03:20:01,951 NEXT SLIDE PLEASE. 5433 03:20:01,951 --> 03:20:03,019 SO THE PATH FOR PROGRESS IS 5434 03:20:03,019 --> 03:20:06,188 FAMILIAR TO ALL THE DISEASE 5435 03:20:06,188 --> 03:20:08,057 GROUPS ON THIS CALL AND IT 5436 03:20:08,057 --> 03:20:09,992 INCLUDED THE ROLE OF THE 5437 03:20:09,992 --> 03:20:12,461 FOUNDATION ORGANIZING PATIENTS, 5438 03:20:12,461 --> 03:20:12,995 MOTIVATING INVESTIGATORS, 5439 03:20:12,995 --> 03:20:14,230 PROVIDING SEED FUND THAGOREAN 5440 03:20:14,230 --> 03:20:16,098 IDENTIFIED THE GENE, PROTEIN, 5441 03:20:16,098 --> 03:20:17,733 PATHWAY AND ULTIMATELY THE 5442 03:20:17,733 --> 03:20:19,235 THERAPEUTIC TARGET OF COURSE IN 5443 03:20:19,235 --> 03:20:21,103 CONCERT WITH NIH STUDIES AND 5444 03:20:21,103 --> 03:20:23,305 FOLLOWED BY PRECLINICAL STUDIES, 5445 03:20:23,305 --> 03:20:25,875 PHASE 1-2 TRIALS AND A PIVOTAL 5446 03:20:25,875 --> 03:20:27,243 TRIAL THAT IDENTIFIED AN 5447 03:20:27,243 --> 03:20:31,647 EFFECTIVE THERAPY. 5448 03:20:31,647 --> 03:20:33,349 NEXT SLIDE, PLEASE. 5449 03:20:33,349 --> 03:20:36,218 EARLY LAM FOUNDATION RESOURCES 5450 03:20:36,218 --> 03:20:37,420 ARE SHOWN HERE. 5451 03:20:37,420 --> 03:20:40,222 THE LAM CLINIC NETWORK HAS BEEN 5452 03:20:40,222 --> 03:20:41,257 CONDUCTING QUARTERLY CASE 5453 03:20:41,257 --> 03:20:42,525 MANAGEMENT CALLS AND COOPERATIVE 5454 03:20:42,525 --> 03:20:44,193 STUDIES FOR OVER A DECADE AND AS 5455 03:20:44,193 --> 03:20:45,694 I MENTIONED WAS THE PLATFORM FOR 5456 03:20:45,694 --> 03:20:48,431 THE RARE LUNG DEC CONSORTIUM, 5457 03:20:48,431 --> 03:20:50,132 THE NHLBI REG TREE AND 5458 03:20:50,132 --> 03:20:51,434 INTRAMURAL PROGRAM HAS MADE 5459 03:20:51,434 --> 03:20:52,468 SUBSTANTIAL CONTRIBUTIONS TO OUR 5460 03:20:52,468 --> 03:20:53,969 UNDERSTANDING OF LAM AND DEFINE 5461 03:20:53,969 --> 03:20:55,838 THE NATURAL HISTORY OF THE 5462 03:20:55,838 --> 03:20:57,540 DISEASE REVEALING THAT THE 5463 03:20:57,540 --> 03:20:59,375 AVERAGE RATE OF LUNG FUNCTION 5464 03:20:59,375 --> 03:21:01,710 DECLINE IS ABOUT 90-MILLILITERS 5465 03:21:01,710 --> 03:21:03,879 IN FEV1 PER YEAR WHICH IS ABOUT 5466 03:21:03,879 --> 03:21:05,948 3 TIMES NORMAL AND MEDIAN 5467 03:21:05,948 --> 03:21:07,716 SURVIVAL FROM TIME OF DIAGNOSIS 5468 03:21:07,716 --> 03:21:10,453 OF OVER 20 YEARS, PROBABLY ON 5469 03:21:10,453 --> 03:21:12,321 THE ORDER OF 25 YEARS. 5470 03:21:12,321 --> 03:21:14,457 LAM LIAISONS ARE A PATIENT 5471 03:21:14,457 --> 03:21:17,893 NETWORK THAT CONVENE REGIONAL 5472 03:21:17,893 --> 03:21:19,728 MEETINGS AND ADVISE THE 5473 03:21:19,728 --> 03:21:20,696 FOWNATION FOR PRIORITIES 5474 03:21:20,696 --> 03:21:21,063 RESEARCH. 5475 03:21:21,063 --> 03:21:21,630 NEXT SLIDE, PLEASE. 5476 03:21:21,630 --> 03:21:23,365 SO A LITTLE BIT MORE ABOUT LAM, 5477 03:21:23,365 --> 03:21:26,502 IT'S A LOW GRADE HORMONEALLY 5478 03:21:26,502 --> 03:21:27,703 RESPONSIVE NEUROECTODERMALY 5479 03:21:27,703 --> 03:21:29,872 PLASM THAT CAUSES SYSTEMIC 5480 03:21:29,872 --> 03:21:31,340 DISSTRUKS OF THE LYMPHATICS IN 5481 03:21:31,340 --> 03:21:31,907 THE LUNG. 5482 03:21:31,907 --> 03:21:32,408 SHOWN IN THIS FORMULARY 5483 03:21:32,408 --> 03:21:33,442 REFERENCE FILE IN THE LOWER LEFT 5484 03:21:33,442 --> 03:21:40,416 OF THE CT OF THE LUNG IN THE 5485 03:21:40,416 --> 03:21:41,016 UPPER RIGHT, [INDISCERNIBLE] 5486 03:21:41,016 --> 03:21:42,618 SHOWN IN THE PANEL IN THE 5487 03:21:42,618 --> 03:21:44,520 MIDRIGHT, AND THE MILKY FLUID 5488 03:21:44,520 --> 03:21:46,288 THAT APPEARS WHEN THE CHEST IS 5489 03:21:46,288 --> 03:21:51,127 TAPPED, SHOWN ON THE INSET, THE 5490 03:21:51,127 --> 03:21:53,796 GENES WERE IDENTIFIED IN 5491 03:21:53,796 --> 03:21:55,297 PATIENTS WITH TUBELER O 5492 03:21:55,297 --> 03:21:57,933 CLEROSEIS WHICH WAS SHOWN IN LAM 5493 03:21:57,933 --> 03:22:00,669 BY ELIZABETH [INDISCERNIBLE] AND 5494 03:22:00,669 --> 03:22:03,539 THE mTOR PATHWAY AND MOLECULAR 5495 03:22:03,539 --> 03:22:05,374 TARGETS WERE WORKED OUT IN FRUIT 5496 03:22:05,374 --> 03:22:07,143 FLIES AND BY [INDISCERNIBLE] AND 5497 03:22:07,143 --> 03:22:09,512 OTHERS INVESTIGATORS, THE mTOR 5498 03:22:09,512 --> 03:22:12,114 PATHWAY IS CENTRAL TO CELLULAR 5499 03:22:12,114 --> 03:22:14,517 METABOLISM, INCLUDING CELL 5500 03:22:14,517 --> 03:22:15,718 PROLIFERATION, AND LIMP ANG O 5501 03:22:15,718 --> 03:22:17,319 GENESIS, AND AT THE TIME OF 5502 03:22:17,319 --> 03:22:20,022 THESE FINDINGS THERE WERE 2 ORAL 5503 03:22:20,022 --> 03:22:21,190 INHIB THORS UTRORS APPROVED FOR 5504 03:22:21,190 --> 03:22:22,725 TRANSPLANT REJECTION THAT WERE 5505 03:22:22,725 --> 03:22:28,631 AVAILABLE FOR PREPURPOSING. 5506 03:22:28,631 --> 03:22:29,431 NEXT SLIDE, PLEASE. 5507 03:22:29,431 --> 03:22:31,400 SO ALTHOUGH A IN LECULAR TARGET 5508 03:22:31,400 --> 03:22:33,736 HAD BEEN IDENTIFIED THERE WERE 5509 03:22:33,736 --> 03:22:34,904 mTOR INHIBITORS AVAILABLE FOR 5510 03:22:34,904 --> 03:22:36,405 REPURPOSING AND A PHASE 1 TRIAL 5511 03:22:36,405 --> 03:22:38,040 HAD SHOWN A PROMISING SIGNAL IN 5512 03:22:38,040 --> 03:22:40,309 THE LUNG AND PATIENTS WERE 5513 03:22:40,309 --> 03:22:41,610 ORGANIZED AND EDUCATED ABOUT THE 5514 03:22:41,610 --> 03:22:43,913 IMPORTANCE OF TRIALS BY THE 5515 03:22:43,913 --> 03:22:44,547 FOUNDATION, NO PHARMACEUTICAL 5516 03:22:44,547 --> 03:22:45,848 FIRM WAS WILLING TO STEP FORWARD 5517 03:22:45,848 --> 03:22:47,716 TO CONDUCT THE TRIAL IN SUCH A 5518 03:22:47,716 --> 03:22:48,284 RARE CONDITION. 5519 03:22:48,284 --> 03:22:50,219 SO A GROUP OF PHYSICIAN 5520 03:22:50,219 --> 03:22:52,955 INVESTIGATORS AROUND THE WORLD 5521 03:22:52,955 --> 03:22:55,824 ORGANIZED TO CONDUCT THIS 5522 03:22:55,824 --> 03:22:56,292 INVESTIGATOR INITIATED 5523 03:22:56,292 --> 03:22:57,293 RANDOMIZED DOUBLE BLIND CONTROL 5524 03:22:57,293 --> 03:23:00,729 TRIAL OF THE mTOR, VERSUS 5525 03:23:00,729 --> 03:23:03,365 PLACEBO IN 89 PATIENTS CALLED 5526 03:23:03,365 --> 03:23:05,100 MILES, THE LAM FOUNDATION 5527 03:23:05,100 --> 03:23:06,936 PROMOTED THE TRIAL AND ASSISTED 5528 03:23:06,936 --> 03:23:07,937 WITH ENROLLMENT. 5529 03:23:07,937 --> 03:23:09,438 THEY INVESTED ABOUT HALF THEIR 5530 03:23:09,438 --> 03:23:10,673 NETWORK AT THE TIME FOR THIS 5531 03:23:10,673 --> 03:23:12,775 TRIAL THAT COST WELL OVER 5532 03:23:12,775 --> 03:23:14,343 $5 MILLION, THEY PUT IN OVER A 5533 03:23:14,343 --> 03:23:16,946 HALF MILLION THERE IS AND MANY 5534 03:23:16,946 --> 03:23:18,747 OTHER FUNDERS INCLUDING UC, 5535 03:23:18,747 --> 03:23:20,649 CHILDREN'S HOSPITAL, FDA, 5536 03:23:20,649 --> 03:23:25,187 PFIZER, TUMOR SPECTRUM CLER O 5537 03:23:25,187 --> 03:23:27,790 SCLEROSIS ALLIANCE, NIH, THERE 5538 03:23:27,790 --> 03:23:33,128 WERE 12 SITES AND 3 COUNTRIES. 5539 03:23:33,128 --> 03:23:35,397 UNIVERSITY OF CINCINNATI WAS THE 5540 03:23:35,397 --> 03:23:38,667 HUB, AND THE LAMB FOUNDATION 5541 03:23:38,667 --> 03:23:41,537 PROVED BE MORE FACILE AT 5542 03:23:41,537 --> 03:23:42,805 HANDLING INVOICES SO THEY TOOK 5543 03:23:42,805 --> 03:23:43,639 OVER THAT FUNCTION. 5544 03:23:43,639 --> 03:23:45,207 THE TRIAL TOOK SEVERAL YEARS TO 5545 03:23:45,207 --> 03:23:47,943 ENROLL, OVER 3 YEARS AND WHEN 5546 03:23:47,943 --> 03:23:49,111 RECRUITMENT LAGGED LISA YOUNG IN 5547 03:23:49,111 --> 03:23:52,147 THE LAB IDENTIFIED A DIAGNOSTIC 5548 03:23:52,147 --> 03:23:53,349 BIOMARKER CALLED VEG FD IN THE 5549 03:23:53,349 --> 03:23:56,385 LOWER RIGHT PANEL THAT ENABLED 5550 03:23:56,385 --> 03:23:58,487 TO TO DISCRIMINATE LAM FROM 5551 03:23:58,487 --> 03:23:59,555 OTHER MIMICS AND ALLOWED 5552 03:23:59,555 --> 03:24:00,389 PATIENTS TO ENROLL WITHOUT THE 5553 03:24:00,389 --> 03:24:03,759 NEED FOR A LUNG BIOPSY WHICH 5554 03:24:03,759 --> 03:24:04,660 REALLY BOOSTED ENROLLMENT. 5555 03:24:04,660 --> 03:24:07,129 WHEN DATA WAS ANALYZED IN THE 5556 03:24:07,129 --> 03:24:08,664 PANEL IN THE UPPER RIGHT IT WAS 5557 03:24:08,664 --> 03:24:10,399 ASHES PARENT THE PATIENTS ON 5558 03:24:10,399 --> 03:24:12,201 PLACEBO LOST ABOUT 11% OF THE 5559 03:24:12,201 --> 03:24:13,836 THEIR LUNG FUNCTION IN THE FORM 5560 03:24:13,836 --> 03:24:19,341 OF FEV1 IN THE FIRST YEAR WHILE 5561 03:24:19,341 --> 03:24:22,945 THOSE ON SIROLMOBOIS, IT 5562 03:24:22,945 --> 03:24:23,245 IMPROVEMENT. 5563 03:24:23,245 --> 03:24:25,080 THERE WAS A REDUCTION IN VEG FD. 5564 03:24:25,080 --> 03:24:26,949 IN THE SECOND YEAR WHEN THE 5565 03:24:26,949 --> 03:24:29,251 PLACEBOS WERE HELD, LUNG 5566 03:24:29,251 --> 03:24:31,453 FUNCTION DECLINE RESUMED IN THE 5567 03:24:31,453 --> 03:24:32,154 [INDISCERNIBLE] GROUP PARALLEL 5568 03:24:32,154 --> 03:24:34,023 WITH THAT WITH THE PLACEBO 5569 03:24:34,023 --> 03:24:34,356 GROUP. 5570 03:24:34,356 --> 03:24:37,226 THESE RESULTS WERE PUBLISH 5571 03:24:37,226 --> 03:24:37,559 INDEED 2011. 5572 03:24:37,559 --> 03:24:38,727 IT'S IMPORTANT TO NOTE THAT 5573 03:24:38,727 --> 03:24:40,062 THROUGHOUT THE 3 YEARS THAT 5574 03:24:40,062 --> 03:24:41,096 MILES WAS ENROLLING IT WAS 5575 03:24:41,096 --> 03:24:42,898 ALREADY CLEAR THAT mTOR 5576 03:24:42,898 --> 03:24:45,067 INHIBITORS WERE VERY PROMISING 5577 03:24:45,067 --> 03:24:47,803 FOR LAM AND AVAILABLE BY 5578 03:24:47,803 --> 03:24:48,637 PRESCRIPTION FROM ANY DOCTOR AND 5579 03:24:48,637 --> 03:24:50,739 A TRIP TO THE PHARMACY. 5580 03:24:50,739 --> 03:24:52,408 YET, THESE PATIENTS SIGNED UP 5581 03:24:52,408 --> 03:24:54,343 FOR A 2 YEAR COMMITMENT RISKING 5582 03:24:54,343 --> 03:24:55,811 LOSING 20% OF THEIR LUNG 5583 03:24:55,811 --> 03:24:56,679 FUNCTION WHILE THEY WERE ON THE 5584 03:24:56,679 --> 03:24:59,615 STUDY IF THEY WERE IN THE 5585 03:24:59,615 --> 03:25:00,015 PLACEBO GROUP. 5586 03:25:00,015 --> 03:25:01,784 AND THE REASON THAT WE HAVE AN 5587 03:25:01,784 --> 03:25:04,620 APPROVED THERAPY FOR LAM IS THAT 5588 03:25:04,620 --> 03:25:05,554 THESE COURAGEOUS PATIENTS 5589 03:25:05,554 --> 03:25:06,689 COMMITTED TO THE ENTIRE 2 YEAR 5590 03:25:06,689 --> 03:25:09,425 COURSE OF THE TRIAL INCLUDING 5591 03:25:09,425 --> 03:25:10,959 OBSERVATION YOU'RE ON NO THERAPY 5592 03:25:10,959 --> 03:25:14,330 AND REMAINED ON THE STUDY FOR 5593 03:25:14,330 --> 03:25:24,073 THE DURATION. 5594 03:25:24,073 --> 03:25:24,873 NEXT SLIDE, PLEASE. 5595 03:25:24,873 --> 03:25:33,549 OH, I'M SORRY THAT THAT WAS THE 5596 03:25:33,549 --> 03:25:35,384 WRONG SLIDE. 5597 03:25:35,384 --> 03:25:36,518 THEY DECLINED BASED ON THE 5598 03:25:36,518 --> 03:25:39,054 RARITY OF LAM AND THE WANING 5599 03:25:39,054 --> 03:25:40,589 PATENT LIFE OF THE DRUG, BY THAT 5600 03:25:40,589 --> 03:25:42,558 TIME WE HEARD ABOUT AN 5601 03:25:42,558 --> 03:25:43,292 ALTERNATHWAY TOALL 5602 03:25:43,292 --> 03:25:44,593 THE COMPANY TO CHANGE A LABEL 5603 03:25:44,593 --> 03:25:51,233 FOR THE DRUG CALLED THE 5604 03:25:51,233 --> 03:25:52,735 CITIZEN'S PETITION, THEY DIDN'T 5605 03:25:52,735 --> 03:25:55,771 LIKE THAT PATHWAY BECAUSE 5606 03:25:55,771 --> 03:25:58,807 MILESTONES ARE NOT REDINED SO 5607 03:25:58,807 --> 03:26:02,444 THE FDA THEMSELVES REACHED OUT. 5608 03:26:02,444 --> 03:26:04,213 SO LAM FOUNDATION, PFIZER AND I 5609 03:26:04,213 --> 03:26:11,487 MET WITH THE FDA, TO CHART A 5610 03:26:11,487 --> 03:26:13,122 COURSE FOR SIROLIMUS, FOR ORPHAN 5611 03:26:13,122 --> 03:26:15,290 DRUG, BREAK THROUGH 5612 03:26:15,290 --> 03:26:16,191 DEZITIONINATION AND SUBMISSION 5613 03:26:16,191 --> 03:26:18,427 OF A SUPPLEMENT AT NDA AND WE 5614 03:26:18,427 --> 03:26:20,396 SENT OUR APPLICATION IN ON 5615 03:26:20,396 --> 03:26:26,969 CHRISTMAS EVE ON 2014 AND WE 5616 03:26:26,969 --> 03:26:28,404 RECEIVED APPROVAL IN UNDER 6 5617 03:26:28,404 --> 03:26:28,670 MONTHS. 5618 03:26:28,670 --> 03:26:29,371 NEXT SLIDE, PLEASE. 5619 03:26:29,371 --> 03:26:32,741 WE ARE VERY PROUD TO HAVE 5620 03:26:32,741 --> 03:26:35,043 FRANCIS COLLINS CITE THE LAM 5621 03:26:35,043 --> 03:26:36,245 FOUNDATION AN EXEMPLAR PROGRESS 5622 03:26:36,245 --> 03:26:40,082 FOR IMMUNITY ON THE NIH RARE 5623 03:26:40,082 --> 03:26:46,922 DISEASE DAY, IN 2016. 5624 03:26:46,922 --> 03:26:48,023 NEXT SLIDE, PLEASE. 5625 03:26:48,023 --> 03:26:50,325 FDA APPROVAL TURNED OUT TO BE A 5626 03:26:50,325 --> 03:26:51,727 REMARKABLE CATALYST FOR A PROVAL 5627 03:26:51,727 --> 03:26:53,595 BY THE GOVERNMENTAL AGENCIES IN 5628 03:26:53,595 --> 03:26:54,463 40 OTHER COUNTRIES, INCLUDING 5629 03:26:54,463 --> 03:26:56,131 SOME WHERE THE DRUG IS ONLY 5630 03:26:56,131 --> 03:27:00,068 AVAILABLE IF THEY HAVE 5631 03:27:00,068 --> 03:27:00,702 GOVERNMENTAL APPROVAL. 5632 03:27:00,702 --> 03:27:03,939 IT'S NOW APPROVE INDEED JAPAN, 5633 03:27:03,939 --> 03:27:05,307 RUSSIA, SOUTH KOREA, BRAZIL, 5634 03:27:05,307 --> 03:27:07,709 MANY COUNTRIES IN SOUTH AMERICA. 5635 03:27:07,709 --> 03:27:08,577 HONG KONG, EUROPEAN UNION. 5636 03:27:08,577 --> 03:27:10,412 WE BELIEVE THAT HALF OF ALL 5637 03:27:10,412 --> 03:27:11,847 PATIENTS WITH ACCESS TO THE DRUG 5638 03:27:11,847 --> 03:27:18,787 ARE CURRENTLY TAKING IT. 5639 03:27:18,787 --> 03:27:19,721 NEXT SLIDE, PLEASE. 5640 03:27:19,721 --> 03:27:21,790 IT'S USEFUL TO REVIEW THE KEY 5641 03:27:21,790 --> 03:27:23,425 INGREDIENTS THAT RESULTED IN A 5642 03:27:23,425 --> 03:27:24,526 TARGETED TREATMENT FOR LAM AND I 5643 03:27:24,526 --> 03:27:26,929 THINK NUMBER 1 IS THE POWER OF 5644 03:27:26,929 --> 03:27:29,131 ACADEMIC HEALTH CENTERS, THE 5645 03:27:29,131 --> 03:27:31,099 EXPERTISE AND RESOURCES OF MAJOR 5646 03:27:31,099 --> 03:27:32,367 ACADEMIC HEALTH CENTERS, MAKE 5647 03:27:32,367 --> 03:27:33,435 THIS POSSIBLE, NO DRUG COMPANY 5648 03:27:33,435 --> 03:27:35,971 WAS WILLING TO DO THIS RESEARCH, 5649 03:27:35,971 --> 03:27:41,143 AND IT WAS THROUGH THE ALTRUISM 5650 03:27:41,143 --> 03:27:41,910 OF PHYSICIAN INVESTIGATORS AND 5651 03:27:41,910 --> 03:27:43,378 MANY OTHERS THAT WE WERE ABLE TO 5652 03:27:43,378 --> 03:27:44,079 LAUNCH THIS TRIAL. 5653 03:27:44,079 --> 03:27:46,248 THE COURAGE AND RESOLVE THE 5654 03:27:46,248 --> 03:27:47,749 PATIENTS WHO ORGANIZED TO 5655 03:27:47,749 --> 03:27:48,851 FACILITATE AND FUND RESEARCH AND 5656 03:27:48,851 --> 03:27:54,223 THEN LINED UP FOR TRIALS, 5657 03:27:54,223 --> 03:27:55,257 FUNDING OF EXCELLENT RESEARCH 5658 03:27:55,257 --> 03:27:56,658 PLIEND TO IMMEDIATE RELEVANCE, 5659 03:27:56,658 --> 03:27:58,660 BASIC RESEARCH IN FRUIT FLIES, 5660 03:27:58,660 --> 03:28:01,897 WORMS AND RODENTS REALLY 5661 03:28:01,897 --> 03:28:03,699 IDENTIFIED MOLECULAR TARGET FOR 5662 03:28:03,699 --> 03:28:05,200 THIS DISEASE, THE ALTRUISM OF 5663 03:28:05,200 --> 03:28:07,102 NOT ONLY MEDICAL CAREGIVERS BUT 5664 03:28:07,102 --> 03:28:10,038 INDUSTRY AND REGULATORS, WE HAD 5665 03:28:10,038 --> 03:28:13,575 TREMENDOUS SUPPORT FOR NO 5666 03:28:13,575 --> 03:28:14,776 COMPENSATION FROM PFIZER, FDA 5667 03:28:14,776 --> 03:28:16,178 AND REGULATE ARES THAT HELPED US 5668 03:28:16,178 --> 03:28:18,146 GET TO THE GOAL LINE. 5669 03:28:18,146 --> 03:28:21,216 ASK ACCESS TO FUNDING USUALLY 5670 03:28:21,216 --> 03:28:24,419 AGENCIES ARE NOT HAPPY ABOUT 5671 03:28:24,419 --> 03:28:25,354 SHARING, FUNDING FOR A TRAIL 5672 03:28:25,354 --> 03:28:28,090 LIKE THIS BUT WE HAD I THINK A 5673 03:28:28,090 --> 03:28:29,291 DOZEN FUNDERS FOR THIS TRIAL 5674 03:28:29,291 --> 03:28:30,893 EACH WILLING TO SUPPORT A 5675 03:28:30,893 --> 03:28:33,395 PORTION OF IT AND FINALLY 5676 03:28:33,395 --> 03:28:34,897 GOVERNMENT ADVOCACY THAT 5677 03:28:34,897 --> 03:28:36,365 HAPPENED WELL BEFORE THE TRIAL 5678 03:28:36,365 --> 03:28:37,866 STARTED INCLUDING THE RARE 5679 03:28:37,866 --> 03:28:39,134 DISEASE ACT OF 2002 WHICH 5680 03:28:39,134 --> 03:28:41,603 PROVIDED THE RARE LUNG DISEASE 5681 03:28:41,603 --> 03:28:43,038 INFRASTRUCTURE THAT MADE AN 5682 03:28:43,038 --> 03:28:48,143 INTERNATIONAL INVESTIGATOR 5683 03:28:48,143 --> 03:28:52,614 INITIATED TRIAL IN LAM. 5684 03:28:52,614 --> 03:28:53,382 NEXT SLIDE, PLEASE. 5685 03:28:53,382 --> 03:28:55,684 SO SINCE THE MILES TRIAL, 5686 03:28:55,684 --> 03:28:56,985 THERE'S BEEN CONTINUING 5687 03:28:56,985 --> 03:28:57,252 PROGRESS. 5688 03:28:57,252 --> 03:28:59,121 THE MILES REGISTRY WAS 5689 03:28:59,121 --> 03:29:00,322 ESTABLISHED ABOUT 7 YEARS AGO, 5690 03:29:00,322 --> 03:29:03,091 THIS IS A REAL WORLD OBSERVAL 5691 03:29:03,091 --> 03:29:04,826 REG TREE TO COLLECT LONGITUDINAL 5692 03:29:04,826 --> 03:29:06,461 DATA ON THE NATURAL HISTORY, 5693 03:29:06,461 --> 03:29:10,399 LONG-TERM SAFETY AND EFFICACY OF 5694 03:29:10,399 --> 03:29:11,300 SIROLMUS THERAPY. 5695 03:29:11,300 --> 03:29:12,868 LAM'S FOUNDATION HAS BEEN 5696 03:29:12,868 --> 03:29:14,136 ASIEVING WITH ENROLLMENT FROM 5697 03:29:14,136 --> 03:29:17,773 THIS PROJECT FROM THE BEGINNING 5698 03:29:17,773 --> 03:29:19,841 AND WE SUPPORTED MIDAS AS THE 5699 03:29:19,841 --> 03:29:20,442 FUNDING RAN OUT. 5700 03:29:20,442 --> 03:29:21,944 THE FIRST REPORT WILL BE IN 2025 5701 03:29:21,944 --> 03:29:24,446 AND A QUICK LOOK AT PRELIMINARY 5702 03:29:24,446 --> 03:29:26,515 DATA SHOWS THERE'S REMARKABLE 5703 03:29:26,515 --> 03:29:27,215 STABILITY OF FAV 1 IN PATIENT 5704 03:29:27,215 --> 03:29:37,693 WHO IS ARE TAKING SIROLIMUS 5705 03:29:38,260 --> 03:29:38,527 DRUG. 5706 03:29:38,527 --> 03:29:46,368 WE ARE NOW WORKING ON A TRIAL 5707 03:29:46,368 --> 03:29:50,138 CALLED MILED, AND IF 5708 03:29:50,138 --> 03:29:52,407 PROPHYLACTIC EARLY LOW DOSE 5709 03:29:52,407 --> 03:29:54,142 PREVENTS PROGRESSION TO MORE 5710 03:29:54,142 --> 03:29:56,078 ADVANCED STAGES IN PATIENTS WITH 5711 03:29:56,078 --> 03:29:56,878 MILD LAM. 5712 03:29:56,878 --> 03:29:59,114 WE'VE HAD 10 SITES, THE LAM 5713 03:29:59,114 --> 03:30:01,617 FOUNDATION HAS BEEN PAYING FOR 5714 03:30:01,617 --> 03:30:02,584 TRAVEL AND ASSISTING WITH 5715 03:30:02,584 --> 03:30:03,986 ENROLLMENT AND WE SHOULD HAVE A 5716 03:30:03,986 --> 03:30:05,520 REPORT OUT THIS COMING YEAR. 5717 03:30:05,520 --> 03:30:08,924 OTHER -- IN ADDITION TO THE 5718 03:30:08,924 --> 03:30:11,760 ANNUAL SEMIANNUAL LAM POSEIUM I 5719 03:30:11,760 --> 03:30:15,364 MENTIONED, LAM FOUNDATION, 1 5720 03:30:15,364 --> 03:30:17,332 SLIDE FORWARD -- THE LAM 5721 03:30:17,332 --> 03:30:20,002 FOUNDATION HAS HAD A NUMBER OF 5722 03:30:20,002 --> 03:30:20,869 INNOVATIVE ESPECIALLY 5723 03:30:20,869 --> 03:30:23,271 CONFERENCES INCLUDING THOSE 5724 03:30:23,271 --> 03:30:24,406 DEVOTED TO BIOMARKER, BIOMARKERS 5725 03:30:24,406 --> 03:30:26,074 AND OTHER NICHE AREAS BUT 1 OF 5726 03:30:26,074 --> 03:30:27,609 MY FAVORITES WAS THE PATIENT 5727 03:30:27,609 --> 03:30:28,877 BENEFIT CONFERENCE WHICH BEGAN 5728 03:30:28,877 --> 03:30:31,380 WITH SURVEYS TO ASK PATES WHAT 5729 03:30:31,380 --> 03:30:32,547 THEIR PRIORITIES FOR RESEARCH 5730 03:30:32,547 --> 03:30:33,982 WERE AND A WORD CLOUD WAS 5731 03:30:33,982 --> 03:30:35,050 DEVELOPED TO REPRESENT THE 5732 03:30:35,050 --> 03:30:36,385 FREQUENCY OF WORDS THAT WERE 5733 03:30:36,385 --> 03:30:39,888 REPEATED IN THE SURVEY, 6 FOCUS 5734 03:30:39,888 --> 03:30:41,323 AREAS WERE CHOSEN, EXPERTS AND 5735 03:30:41,323 --> 03:30:43,792 COMPANIES IN THOSE AREAS WERE 5736 03:30:43,792 --> 03:30:47,229 RECRUITED AND 8 PROJECTS WERE 5737 03:30:47,229 --> 03:30:48,296 FUNDED FROM THE 200,000 THERE IS 5738 03:30:48,296 --> 03:30:49,731 THAT WAS RAISED DURING THE 5739 03:30:49,731 --> 03:30:50,298 EVENT. 5740 03:30:50,298 --> 03:30:53,602 THERE ARE STUDIES UNDERWAY TO 5741 03:30:53,602 --> 03:30:55,003 ADDRESS FATIGUE, EXERCISE, 5742 03:30:55,003 --> 03:30:57,739 OXYGEN DELIVERY, COPING AND 5743 03:30:57,739 --> 03:30:58,040 BIOMARKERS. 5744 03:30:58,040 --> 03:30:59,675 THE I THINK THE PATIENT 5745 03:30:59,675 --> 03:31:02,210 COMMUNITY APPRECIATED THAT IN 5746 03:31:02,210 --> 03:31:05,647 ADDITION TO OUR BASIC CLINICAL 5747 03:31:05,647 --> 03:31:05,881 TRIALS. 5748 03:31:05,881 --> 03:31:06,581 FOWBDATION WAS FOCUSING ON THE 5749 03:31:06,581 --> 03:31:08,016 PROBLEMS THAT AFFECT THEIR DAILY 5750 03:31:08,016 --> 03:31:11,486 LIVES. 5751 03:31:11,486 --> 03:31:12,754 NEXT SLIDE, PLEASE. 5752 03:31:12,754 --> 03:31:14,156 THE LAM FOUNDATION AWLINGS 5753 03:31:14,156 --> 03:31:23,031 PROVIDED SEED FUNDING TO GET 5754 03:31:23,031 --> 03:31:23,699 GUIDE LINES STARTED. 5755 03:31:23,699 --> 03:31:25,801 IN THESE GUIDE LINES WE'VE 5756 03:31:25,801 --> 03:31:26,735 INCORPORATED RECOMMENDATIONINGS 5757 03:31:26,735 --> 03:31:30,439 FOR THE USE OF VEG FD AS A 5758 03:31:30,439 --> 03:31:31,940 DIAGNOSTIC BIOMARKER THAT CAN 5759 03:31:31,940 --> 03:31:33,608 OBVIATE THE NEED FOR BIOPSY AND 5760 03:31:33,608 --> 03:31:39,414 THE INDICATIONS FOR THE USE OF 5761 03:31:39,414 --> 03:31:41,016 SIROLIMUS, AND THE DOCUMENTS 5762 03:31:41,016 --> 03:31:41,983 HELP TO DISSEMINATE NEW 5763 03:31:41,983 --> 03:31:43,151 INFORMATION ABOUT MANAGEMENT AND 5764 03:31:43,151 --> 03:31:48,290 TREATMENT. 5765 03:31:48,290 --> 03:31:49,091 NEXT SLIDE, PLEASE. 5766 03:31:49,091 --> 03:31:50,325 FINALLY, THE LAM FOUNDATION 5767 03:31:50,325 --> 03:31:54,029 RECOGNIZED THE POWER OF THE NEW 5768 03:31:54,029 --> 03:31:54,629 SINGLE SRNARE--ADMINISTRATIVE 5769 03:31:54,629 --> 03:31:57,833 SEQ TECHNOLOGY THAT THE LAST 5770 03:31:57,833 --> 03:32:01,103 SPEAKER WAS TALKING ABOUT AND 5771 03:32:01,103 --> 03:32:02,237 FUNDED THE TRANSCRIPTOMIC 5772 03:32:02,237 --> 03:32:03,638 PROFILE OF FROM LUNG PATIENTS 5773 03:32:03,638 --> 03:32:06,041 WHO HAD BEEN TRANSPLANTED. 5774 03:32:06,041 --> 03:32:08,343 WHEN THE LUNG WAS DISASSOCIATED 5775 03:32:08,343 --> 03:32:10,445 INTO INDIVIDUAL CELLS THEY WERE 5776 03:32:10,445 --> 03:32:11,379 CLUSTERED ON SIGNATURES IT 5777 03:32:11,379 --> 03:32:13,248 BECAME APPARENT THERE WAS AN 5778 03:32:13,248 --> 03:32:14,416 ECTOPIC POPULATION THAT'S NEVER 5779 03:32:14,416 --> 03:32:16,651 BEEN IDENTIFIED IN THE LUNG 5780 03:32:16,651 --> 03:32:17,085 BEFORE. 5781 03:32:17,085 --> 03:32:18,920 WHEN THAT SIGNATURE WAS BLASTED 5782 03:32:18,920 --> 03:32:19,888 AGAINST ALL OTHER ORGANS IN THE 5783 03:32:19,888 --> 03:32:21,857 BODY ISSUES THE PROFILE 5784 03:32:21,857 --> 03:32:24,192 OVERLAPPED REMARKABLY WITH THAT 5785 03:32:24,192 --> 03:32:29,898 OF THE U TERUS, IN ADDITION THE 5786 03:32:29,898 --> 03:32:30,799 TUBEEROUS MUTATION IN THE LUNG 5787 03:32:30,799 --> 03:32:32,934 OF 1 OF THE PATIENTS WERE DEMON 5788 03:32:32,934 --> 03:32:36,738 TRAITED TO BE IDENTICAL 5789 03:32:36,738 --> 03:32:39,107 DEMONSTRATING THE SOIRS AS THE 5790 03:32:39,107 --> 03:32:41,443 CELL FROM THE UTERUS THAT 5791 03:32:41,443 --> 03:32:42,677 DESTROYS THE LUNG. 5792 03:32:42,677 --> 03:32:48,884 AND FOR IPF THESE ARE PUBLICLY 5793 03:32:48,884 --> 03:32:50,752 AVAILABLE IN THE ATLAS. 5794 03:32:50,752 --> 03:32:51,853 NEXT SLIDE, PLEASE. 5795 03:32:51,853 --> 03:32:53,555 SO JUST REVIEWING LAM FOUNDATION 5796 03:32:53,555 --> 03:32:55,957 ROLES AND LAM PROGRESS, LAM 5797 03:32:55,957 --> 03:32:57,592 FOUNDATION ORGANIZED EDUCATED 5798 03:32:57,592 --> 03:32:58,860 MOBILIZED PATIENTS, CONDUCTED 5799 03:32:58,860 --> 03:32:59,928 ANNUAL PATIENT INVESTIGATOR 5800 03:32:59,928 --> 03:33:03,098 CONFERENCES, THAT GAVE HOPE TO 5801 03:33:03,098 --> 03:33:04,166 PATIENTS AND INSPIRATION TO 5802 03:33:04,166 --> 03:33:06,067 SCIENTISTS, IT'S HARD TO 5803 03:33:06,067 --> 03:33:08,570 OVERSTATE HOW MUCH THAT KIND OF 5804 03:33:08,570 --> 03:33:09,404 INSPIRATION CONTRIBUTED TO THE 5805 03:33:09,404 --> 03:33:13,341 COMMITMENT OF OUR INVESTIGATORS. 5806 03:33:13,341 --> 03:33:15,177 THEY DEVELOPED THE LAM CLINIC 5807 03:33:15,177 --> 03:33:17,846 NETWORK TO FOCUS REFERRALS AND 5808 03:33:17,846 --> 03:33:19,247 NURTURE EXPERTISE AND FACILITATE 5809 03:33:19,247 --> 03:33:20,849 RESEARCH, DEVELOP A PEER 5810 03:33:20,849 --> 03:33:21,683 REVIEWED SCIENTIFIC RESEARCH 5811 03:33:21,683 --> 03:33:24,219 PROGRAM, THAT FORM THE BASIS FOR 5812 03:33:24,219 --> 03:33:25,520 TRIALS, PARTNER INDEED LARGE 5813 03:33:25,520 --> 03:33:27,722 GRANT INITTIAIVE ITS SUCH AS THE 5814 03:33:27,722 --> 03:33:29,925 NIH FOR A LUNG DEC CONSORTIUM, 5815 03:33:29,925 --> 03:33:32,661 PLAYED KEY ROLE IN TRIAL FUND 5816 03:33:32,661 --> 03:33:34,796 NOTHING THE CONDUCT OF 5817 03:33:34,796 --> 03:33:36,598 RANDOMIZED TRIALS INCLUDING 5818 03:33:36,598 --> 03:33:37,699 MILES AND MILED. 5819 03:33:37,699 --> 03:33:39,367 AND RESULTED IN BIOMARKER AND 5820 03:33:39,367 --> 03:33:40,302 EFFECTIVE TREATMENT. 5821 03:33:40,302 --> 03:33:42,137 IT FUNDED EARLY STUDIES 5822 03:33:42,137 --> 03:33:43,104 IDENTIFYING THE METASTATIC 5823 03:33:43,104 --> 03:33:46,274 MECHANISMS AND RECENT STUDIES 5824 03:33:46,274 --> 03:33:47,309 IDENTIFYING THE TERROUS OF THE 5825 03:33:47,309 --> 03:33:49,945 SOURCE OF THE CELL THAT DESTROYS 5826 03:33:49,945 --> 03:33:51,880 THE LUNG, FUNDED PRACTICAL GUIDE 5827 03:33:51,880 --> 03:33:54,749 LINES AND PATIENT CONFERENCES 5828 03:33:54,749 --> 03:33:57,319 AND ENDOWED A CHAIR FOR 5829 03:33:57,319 --> 03:33:57,986 SUSTAINABLE SCIENTIFIC COUNSELS 5830 03:33:57,986 --> 03:34:02,624 AND I WILL ADVOCACY WITH PHARMA, 5831 03:34:02,624 --> 03:34:03,625 FDA, NIH AND CONGRESS. 5832 03:34:03,625 --> 03:34:04,893 NEXT SLIDE ISSUES PLEASE. 5833 03:34:04,893 --> 03:34:06,094 THERE ARE MANY PEOPLE TO THANK 5834 03:34:06,094 --> 03:34:09,464 AND ONLY A FEW OF THEM ARE 5835 03:34:09,464 --> 03:34:11,733 REPRESENTED HERE, BUT CO 5836 03:34:11,733 --> 03:34:14,502 INVESTIGATORS AND MULTIPLE 5837 03:34:14,502 --> 03:34:17,138 TRIALS, LAM FOUNDATION, MILES PI 5838 03:34:17,138 --> 03:34:18,673 AND SITE TEAMS, OUR UNIVERSITY 5839 03:34:18,673 --> 03:34:20,408 AND CHILDREN'S HOSPITAL SUPPORT, 5840 03:34:20,408 --> 03:34:23,778 FDA FOR THEIR HELP WITH GETTING 5841 03:34:23,778 --> 03:34:25,413 THE APPROVAL OF THE DRUG 5842 03:34:25,413 --> 03:34:28,583 COMPLETED AND A LOT OF HELP FROM 5843 03:34:28,583 --> 03:34:32,220 PFIZER FROM THE VERY BEGINNING 5844 03:34:32,220 --> 03:34:34,189 PROVIDING THE DRUG THROUGH 5845 03:34:34,189 --> 03:34:37,025 PURSUING COMPLETE FDA APPROVAL 5846 03:34:37,025 --> 03:34:37,259 WITH US. 5847 03:34:37,259 --> 03:34:40,095 THANK YOU VERY MUCH. 5848 03:34:40,095 --> 03:34:42,163 >> THANK YOU DR. Mc CORMACK, 5849 03:34:42,163 --> 03:34:43,198 LOOK FORWARD TO TALKING TO YOU 5850 03:34:43,198 --> 03:34:44,432 AS WELL DURING THE PANELMENT SO 5851 03:34:44,432 --> 03:34:47,602 NEXT WE ARE GOING TO HAVE THE 5852 03:34:47,602 --> 03:34:51,439 BRIDGE TO A PANEL DISCUSSION. 5853 03:34:51,439 --> 03:34:53,675 AND DR. CASE WILL SPEND A FEW 5854 03:34:53,675 --> 03:34:55,377 MINUTES MAKING THAT BRIDGE 5855 03:34:55,377 --> 03:35:03,785 BEFORE WE BRING EVERYONE BACK 5856 03:35:03,785 --> 03:35:05,553 FOR THE DISCUSSION. 5857 03:35:05,553 --> 03:35:06,388 >> THANK YOU MARRAH, APPRECIATE 5858 03:35:06,388 --> 03:35:06,755 THAT. 5859 03:35:06,755 --> 03:35:08,690 I JUST WANTED TO TAKE A FEW 5860 03:35:08,690 --> 03:35:13,161 MINUTES AND NOT NECESSARILY 5861 03:35:13,161 --> 03:35:15,697 REVISIT ALL OF THE GREAT 5862 03:35:15,697 --> 03:35:17,799 INITIATIVES AND INFORMATION THAT 5863 03:35:17,799 --> 03:35:20,035 OUR OTHER PANELISTS HAVE TALKED 5864 03:35:20,035 --> 03:35:21,903 ABOUT BUT SORT OF ECHO A FEW 5865 03:35:21,903 --> 03:35:24,973 THINGS THAT HAVE SOME MICE 5866 03:35:24,973 --> 03:35:27,676 OVERLAP WITH THE PULMONARY 5867 03:35:27,676 --> 03:35:28,176 FIBROSIS FOUNDATION. 5868 03:35:28,176 --> 03:35:29,177 BEFORE I GET STARTED I WOULD 5869 03:35:29,177 --> 03:35:30,912 LIKE TO THANK THE COMMITTEE FOR 5870 03:35:30,912 --> 03:35:34,382 THIS OPPORTUNITY TO SPEAK WITH 5871 03:35:34,382 --> 03:35:36,518 EVERYONE AND TO SPEAK WITH OUR 5872 03:35:36,518 --> 03:35:37,285 PANELISTS TODAY. 5873 03:35:37,285 --> 03:35:39,721 I WANTED TO SHARE A LITTLE BIT 5874 03:35:39,721 --> 03:35:40,689 ABOUT THE PF FOUNDATION AND SOME 5875 03:35:40,689 --> 03:35:42,590 OF THE WAYS THAT ARE EXPERIENCE 5876 03:35:42,590 --> 03:35:44,225 JUST REFLECTS THOSE IMPORTANT 5877 03:35:44,225 --> 03:35:45,994 WAYS THAT PATIENT ADVOCACY 5878 03:35:45,994 --> 03:35:47,295 ORGANIZATIONS CAN SUPPORT 5879 03:35:47,295 --> 03:35:51,299 THERAPEUTIC DEVELOPMENT. 5880 03:35:51,299 --> 03:35:53,034 NEXT SLIDE, PLEASE. 5881 03:35:53,034 --> 03:35:57,138 SO THE PFF IS A PATIENT ADVOCACY 5882 03:35:57,138 --> 03:35:58,306 NONPROFIT ORGANIZE EGG, 5883 03:35:58,306 --> 03:36:03,378 DEDICATED TO ADVANCING CARE OF 5884 03:36:03,378 --> 03:36:04,679 PEOPLE WITH PULMONARY FIBROSIS 5885 03:36:04,679 --> 03:36:07,082 AND OUR MISSION IS REFLECTED IN 5886 03:36:07,082 --> 03:36:10,218 THE SIGNATURE PROGRAMS, I NOT GO 5887 03:36:10,218 --> 03:36:12,921 INTO DEPTH OF ALL THINGS WE DO 5888 03:36:12,921 --> 03:36:14,522 AT THE PULMONARY FIBROSIS 5889 03:36:14,522 --> 03:36:16,891 FOUNDATION BUT I HIGHLIGHT A FEW 5890 03:36:16,891 --> 03:36:17,792 OF OUR PROGRAMS. 5891 03:36:17,792 --> 03:36:20,261 ONE OF THOSE PARTS OF THE 5892 03:36:20,261 --> 03:36:21,930 MISSION ACCELERATING NEW 5893 03:36:21,930 --> 03:36:23,331 THERAPEUTIC DEVELOPMENT INCLUDES 5894 03:36:23,331 --> 03:36:24,499 PROGRAMS AND INVESTMENTS INTO 5895 03:36:24,499 --> 03:36:27,168 EARLY CAREER 5896 03:36:27,168 --> 03:36:30,271 INVESTIGATORS, THROUGH OUR 5897 03:36:30,271 --> 03:36:30,805 PFF SCHOLARS PROGRAM, OUR 5898 03:36:30,805 --> 03:36:32,574 REGISTRY WHICH I WILL TALK A 5899 03:36:32,574 --> 03:36:34,309 LITTLE BIT MORE ABOUT IN DEPTH 5900 03:36:34,309 --> 03:36:36,177 AND OUR WORK SUPPORTING CLINICAL 5901 03:36:36,177 --> 03:36:39,381 TRIAL EDUCATION AND RECRUITMENT. 5902 03:36:39,381 --> 03:36:41,082 ANOTHER PILLAR OF THAT MISSION 5903 03:36:41,082 --> 03:36:42,817 IS ADVANCING QUALITY OF CARE 5904 03:36:42,817 --> 03:36:45,320 THROUGHEUR CARE CENTER NETWORK, 5905 03:36:45,320 --> 03:36:49,057 IT WAS FOUNDED IN 2014 WITH JUST 5906 03:36:49,057 --> 03:36:51,860 A HANDFUL OFITES BUT NOW CLIN 5907 03:36:51,860 --> 03:36:53,328 CLUEDS MORE THAN 80 U.S. BAS 5908 03:36:53,328 --> 03:36:55,864 CENTERS IN THE VAST MAJORITY OF 5909 03:36:55,864 --> 03:37:00,902 ESTATES WE DON'T REACH ALL OF BO 5910 03:37:00,902 --> 03:37:03,638 EXPANSION OF A CLINICAL 5911 03:37:03,638 --> 03:37:04,773 ASSOCIATES PARTNER NETWORK THAT 5912 03:37:04,773 --> 03:37:07,175 IS MORE OUTSIDE OF LARMINGER 5913 03:37:07,175 --> 03:37:10,945 ACADEMIC INSTITUTIONS AND 5914 03:37:10,945 --> 03:37:15,417 REACHES INTO SOME OF THE SMALLER 5915 03:37:15,417 --> 03:37:16,284 COMMUNITY BASED PRACTICES THAT 5916 03:37:16,284 --> 03:37:17,886 CARE FOR SO MANY OF OUR PATIENTS 5917 03:37:17,886 --> 03:37:19,554 THAT DON'T COME TO LARGER 5918 03:37:19,554 --> 03:37:19,888 CENTERS. 5919 03:37:19,888 --> 03:37:21,056 AND FINALLY WE BUILD COMMUNITY 5920 03:37:21,056 --> 03:37:23,558 WITH OUR SUPPORT PROGRAM ANDS 5921 03:37:23,558 --> 03:37:27,162 EDUCATIONAL PROGRAMS, INCLUDING 5922 03:37:27,162 --> 03:37:31,066 OUR SERIES, WE HAVE A 5923 03:37:31,066 --> 03:37:32,233 LOT OF EDUCATIONAL CONTENT ON 5924 03:37:32,233 --> 03:37:33,301 OUR WEBSITE AND THOSE PRINTED 5925 03:37:33,301 --> 03:37:35,136 MATERIALS THAT ARE GOING OUT TO 5926 03:37:35,136 --> 03:37:39,674 SUPPORT GROUPS AND TO CLINICS. 5927 03:37:39,674 --> 03:37:41,876 ASK OUR BI-ANNUAL PULMONARY 5928 03:37:41,876 --> 03:37:43,878 FIBROSIS SUMMIT THAT INCLUDES 5929 03:37:43,878 --> 03:37:44,712 PATIENTS, CAREGIVERS, HEALTHCARE 5930 03:37:44,712 --> 03:37:45,814 PROFESSIONALS AND INDUSTRY 5931 03:37:45,814 --> 03:37:49,551 PARTNERS WHICH IS A REALLY 5932 03:37:49,551 --> 03:37:54,089 UNIQUE OPPORTUNITY FOR THOSE 5933 03:37:54,089 --> 03:37:56,658 PARTIES TO ALL BE TOGETHER IN AN 5934 03:37:56,658 --> 03:37:58,326 EDUCATION AND NETWORKING 5935 03:37:58,326 --> 03:37:59,260 CAPACITY. 5936 03:37:59,260 --> 03:38:01,096 NEXT SLIDE PLEASE. 5937 03:38:01,096 --> 03:38:03,598 THE PULMONARY FIBROSIS 5938 03:38:03,598 --> 03:38:06,134 FOUNDATION'S PATIENT REGISTRY 5939 03:38:06,134 --> 03:38:08,169 BEGAN PLANNING YEARS BEFORE THE 5940 03:38:08,169 --> 03:38:17,112 FDA APPROVAL OF THE 2 EXISTING 5941 03:38:17,112 --> 03:38:18,146 ANTIFIBROTIC THERAPIES, AND ALL 5942 03:38:18,146 --> 03:38:19,547 THE PLANNING AND MORE FUNDING 5943 03:38:19,547 --> 03:38:21,282 THAT BECAME AVAILABLE AFTER THE 5944 03:38:21,282 --> 03:38:22,817 ADVENT OF THOSE THERAPIES 5945 03:38:22,817 --> 03:38:27,088 LAUNCHED INUN 2016 AND RECRUITA 5946 03:38:27,088 --> 03:38:31,759 ITS GOAL OF 2000 SUBJEKS IN A 5947 03:38:31,759 --> 03:38:33,561 CENTER-BASED FASHION FROM 42 5948 03:38:33,561 --> 03:38:35,163 EXISTING CARE CENTER SITES AT 5949 03:38:35,163 --> 03:38:37,465 THAT TIME. 5950 03:38:37,465 --> 03:38:39,801 THE SUBJECTS INCLUDED ARE VERY 5951 03:38:39,801 --> 03:38:40,735 WELL CHARACTERIZED, CLINICALLY, 5952 03:38:40,735 --> 03:38:42,971 THELI DATA COLLECTION WENT ON FR 5953 03:38:42,971 --> 03:38:47,308 OVER 5 YEARS, AND INCLUDES HIGH 5954 03:38:47,308 --> 03:38:48,476 RESOLUTION CT IMAGING, BLOOD 5955 03:38:48,476 --> 03:38:51,713 SAMPLES AT BASE LINE, CEREAL 5956 03:38:51,713 --> 03:38:54,249 PULMONARY FUNCTION TESTS AND 5957 03:38:54,249 --> 03:38:55,083 PATIENT REPORTED OUTCOMES 5958 03:38:55,083 --> 03:38:57,085 REPORTED AS WELL. 5959 03:38:57,085 --> 03:38:59,187 THE REGISTRY SOUGHT DATA 5960 03:38:59,187 --> 03:39:03,191 THAT 5 + YEAR TIME PERIOD WE DID 5961 03:39:03,191 --> 03:39:08,363 SEE OF COURSE ATTRITION BASED ON 5962 03:39:08,363 --> 03:39:10,231 THE MORTALITY OF PULMONARY 5963 03:39:10,231 --> 03:39:13,434 FIBROSIS, BUT TO DATE, THE DATA 5964 03:39:13,434 --> 03:39:14,936 HAS BEEN INCREDIBLY VALUABLE, 5965 03:39:14,936 --> 03:39:18,039 IT'S BEEN USED FOR MORE THAN 50 5966 03:39:18,039 --> 03:39:19,908 DIFFERENT RESEARCH STUDIES, WE 5967 03:39:19,908 --> 03:39:21,376 HAVE OTHERS THAT ARE IN PROGRESS 5968 03:39:21,376 --> 03:39:23,077 AND ACADEMIC AS WELL AS INDUSTRY 5969 03:39:23,077 --> 03:39:24,579 PARTNERS THAT ARE STILL 5970 03:39:24,579 --> 03:39:27,415 INTERESTED IN UTILIZING THAT 5971 03:39:27,415 --> 03:39:29,884 DATA, FOR THEIR DIFFERENT 5972 03:39:29,884 --> 03:39:30,151 PROJECTS. 5973 03:39:30,151 --> 03:39:32,220 MOST OF THE STUDIES THAT HAVE 5974 03:39:32,220 --> 03:39:33,521 BEEN DONE TO DATE ARE CLINICAL 5975 03:39:33,521 --> 03:39:37,258 BUT THE BASIC AND TRANSLATIONAL 5976 03:39:37,258 --> 03:39:38,960 INVESTIGATIONS ARE ALSO VERY 5977 03:39:38,960 --> 03:39:41,696 WELL REPRESENTED. 5978 03:39:41,696 --> 03:39:42,697 NEXT SLIDE, PLEASE. 5979 03:39:42,697 --> 03:39:45,667 I WANT TO HIGHLIGHT 2 PROGRAMS 5980 03:39:45,667 --> 03:39:48,169 THAT HAVE UTILIZED AND BORN 5981 03:39:48,169 --> 03:39:52,874 FROM, THE REGISTRY DATA AND NOT 5982 03:39:52,874 --> 03:39:56,077 INCLUDE THE PRECISIONCI STUDY 5983 03:39:56,077 --> 03:39:58,112 WHICH IS THE FIRST PHARMA CO 5984 03:39:58,112 --> 03:40:01,349 GENOMICS STUDY IN IDIO PATHIC 5985 03:40:01,349 --> 03:40:02,016 PULMONARY FIBROSIS. 5986 03:40:02,016 --> 03:40:04,085 IT TOOK ITS INITIAL RECRUITMENT 5987 03:40:04,085 --> 03:40:08,823 DIRECTLY FROM THE PFF REGISTRY 5988 03:40:08,823 --> 03:40:09,557 REGISTRY 5989 03:40:09,557 --> 03:40:09,924 PARTICIPANTS. 5990 03:40:09,924 --> 03:40:15,330 THOSE WERE PRESCREENED FOR THE 5991 03:40:15,330 --> 03:40:17,398 TILE-UP GENO TYPE OF INTEREST 5992 03:40:17,398 --> 03:40:19,834 AND THE SITES THAT PARTICIPATED 5993 03:40:19,834 --> 03:40:23,538 IN THE REGISTRY WERE THEN ABLE 5994 03:40:23,538 --> 03:40:24,839 TO REACH OUT DIRECTLY TO THE 5995 03:40:24,839 --> 03:40:25,773 SUBJECTS AND RECRUIT THEM 5996 03:40:25,773 --> 03:40:28,810 QUICKLY INTO THE STUDY. 5997 03:40:28,810 --> 03:40:31,246 AND THEN ALSO THE PROLIFIC 5998 03:40:31,246 --> 03:40:32,847 CONSORTIUM THAT DR. NUTTER 5999 03:40:32,847 --> 03:40:34,582 MENTIONED EARLIER WHICH INVOLVES 6000 03:40:34,582 --> 03:40:38,553 16 INDUSTRY PARTNERS ISSUES THE 6001 03:40:38,553 --> 03:40:40,255 PULMONARY FIBROSIS FOUNDATION 6002 03:40:40,255 --> 03:40:43,725 AND 3 LAKES FOUNDATION TO USE 6003 03:40:43,725 --> 03:40:46,661 THE REG TREE TO IDENTIFY 6004 03:40:46,661 --> 03:40:47,729 CIRCULATING PULMONARY MARKERS 6005 03:40:47,729 --> 03:40:52,533 AND FIBROSIS. 6006 03:40:52,533 --> 03:40:54,636 NEXT SLIDE, PLEASE. 6007 03:40:54,636 --> 03:40:56,838 IN ADDITION TO THE SCIENTIFIC 6008 03:40:56,838 --> 03:40:59,674 WORK AND THE REGISTRY, AS THAT 6009 03:40:59,674 --> 03:41:00,808 VALUABLE TOOL, I WANT TO 6010 03:41:00,808 --> 03:41:02,810 HIGHLIGHT SOME OF THE WORK WE DO 6011 03:41:02,810 --> 03:41:07,248 IN EDUCATION AS WELL AS 6012 03:41:07,248 --> 03:41:08,283 RECRUITMENT FOR CLINICAL TRIALS. 6013 03:41:08,283 --> 03:41:15,256 WE CAN DO ALL OF THAT AND ALL 6014 03:41:15,256 --> 03:41:17,225 OUR PATIENTS TO PARTICIPATE IN 6015 03:41:17,225 --> 03:41:18,526 THOSE CLINICAL TRIALS AND AS WE 6016 03:41:18,526 --> 03:41:20,028 CAN SEE THEY DON'T GO ANYWHERE, 6017 03:41:20,028 --> 03:41:22,797 SO WE'RE VERY FORTUNATE TO HAVE 6018 03:41:22,797 --> 03:41:24,198 A ROBUST PIPELINE, BUT WE'RE 6019 03:41:24,198 --> 03:41:27,101 RECRUITING FOR A RELATIVELY 6020 03:41:27,101 --> 03:41:28,870 SMALL POPULATION, WITH LOTS OF 6021 03:41:28,870 --> 03:41:32,307 BARRIERS TO TRIAL ENROLLMENT AND 6022 03:41:32,307 --> 03:41:36,311 SO, WE DO HAVE A ROBUST SUITE OF 6023 03:41:36,311 --> 03:41:37,712 EDUCATIONAL OFFERINGS AT THE 6024 03:41:37,712 --> 03:41:38,279 FOUNDATION INCLUDING THOSE 6025 03:41:38,279 --> 03:41:40,081 SPECIFIC TO RESEARCH AND 6026 03:41:40,081 --> 03:41:41,382 CLINICAL TRIALS. 6027 03:41:41,382 --> 03:41:44,185 OUR WEBSITE HAS A CLINICAL 6028 03:41:44,185 --> 03:41:46,220 TRIALS EDUCATION CENTER, WHERE 6029 03:41:46,220 --> 03:41:49,090 PATIENTS CAN FIND INFORMATION 6030 03:41:49,090 --> 03:41:49,757 ABOUT CLINICAL TRIALS 6031 03:41:49,757 --> 03:41:54,128 THEMSELVES, WHAT'S IT LIKE TO 6032 03:41:54,128 --> 03:41:56,964 PARTICIPATE AND THEN ALSO 6033 03:41:56,964 --> 03:41:57,598 INFORMATION ABOUT SPECIFIC 6034 03:41:57,598 --> 03:41:57,965 CLINICAL TRIALS. 6035 03:41:57,965 --> 03:41:59,767 WE HAVE A PIPELINE THAT IS KEPT 6036 03:41:59,767 --> 03:42:01,703 UP TO DATE ON THIS SITE WHERE 6037 03:42:01,703 --> 03:42:07,175 THEY CAN GO AND LOOK AT WHAT IS 6038 03:42:07,175 --> 03:42:08,109 IN DEVELOPMENT AND FIND 6039 03:42:08,109 --> 03:42:09,510 INFORMATION ON STUDIES OF 6040 03:42:09,510 --> 03:42:12,513 INTEREST TO THEM. HAVE WRITTENAT 6041 03:42:12,513 --> 03:42:14,982 CAN BE USED I CLINIC, I USE 6042 03:42:14,982 --> 03:42:16,484 THIS REGULARLY, TO EDUCATE MY 6043 03:42:16,484 --> 03:42:19,721 PATIENTS ON CLINICAL TRIALS AND 6044 03:42:19,721 --> 03:42:21,622 PARTICIPATION AS WE'REALKING 6045 03:42:21,622 --> 03:42:23,324 ABOUT SCREENING AND ENROLLMENT, 6046 03:42:23,324 --> 03:42:25,326 AND THEN WE HAVE DIDACTIC 6047 03:42:25,326 --> 03:42:26,828 TEACHING OFFERINGS THROUGH OUR 6048 03:42:26,828 --> 03:42:29,130 WEBINARS AND DURING OUR 6049 03:42:29,130 --> 03:42:31,165 IN-PERSON EDUCATION AT THE 6050 03:42:31,165 --> 03:42:38,039 BI-ANNUAL SUMMIT. 6051 03:42:38,039 --> 03:42:39,607 NEXT SLIDE, PLEASE. 6052 03:42:39,607 --> 03:42:41,275 AND THEN FINALLY I'LL MENTION 6053 03:42:41,275 --> 03:42:42,744 OUR CLINICAL TRIAL FINDER WHICH 6054 03:42:42,744 --> 03:42:44,812 IS IN PARTNERSHIP WITH THE CARE 6055 03:42:44,812 --> 03:42:45,747 BOX PLATFORM, IT ALLOWS PATIENTS 6056 03:42:45,747 --> 03:42:47,582 TO GO IN AND CREATE A PROFILE 6057 03:42:47,582 --> 03:42:49,083 THEY CAN ENTER THENT OWN 6058 03:42:49,083 --> 03:42:53,454 THEY CAN ACTUALLY MATCH TOL THEL 6059 03:42:53,454 --> 03:42:55,189 STUDIES WITHIN A CERTAIN RADIUS, 6060 03:42:55,189 --> 03:42:56,824 FOR WHICH THEY MIGHT BE 6061 03:42:56,824 --> 03:43:01,429 ELIGIBLE, AND THEN IT PROVIDES 6062 03:43:01,429 --> 03:43:03,264 SUPPORT TO INTERESTED PATIENTS 6063 03:43:03,264 --> 03:43:04,465 VIA E-MAIL AND TELEPHONES. 6064 03:43:04,465 --> 03:43:06,834 THEY CAN REACH A LIVE PERSON, 6065 03:43:06,834 --> 03:43:11,639 MOST OF THE PEOPLE WHO REACH OUT 6066 03:43:11,639 --> 03:43:13,641 TO THE PLATFORM HAVE MULTIPLE 6067 03:43:13,641 --> 03:43:15,143 CONTACTS FOR CLARIFYING AND WITH 6068 03:43:15,143 --> 03:43:17,211 HELP FOR REACHING OUT TO STUDY 6069 03:43:17,211 --> 03:43:21,582 SITES IF IT'S NEEDED. 6070 03:43:21,582 --> 03:43:22,083 NEXT SLIDE,E. 6071 03:43:22,083 --> 03:43:25,653 SO BEFORE WE GET TO OUR PANEL, I 6072 03:43:25,653 --> 03:43:27,088 WANT TO JUST LASTLY HIGHLIGHT 6073 03:43:27,088 --> 03:43:29,924 THE IMPORTANCE OF PATIENT 6074 03:43:29,924 --> 03:43:31,526 PARTICIPATION AND CAREGIVER 6075 03:43:31,526 --> 03:43:33,828 SUPPORT AT ALL POINTS IN THAT 6076 03:43:33,828 --> 03:43:36,998 CLINICAL TRIAL PROCESS, WE 6077 03:43:36,998 --> 03:43:38,433 UNDERSTAND THAT AGAIN, THERE ARE 6078 03:43:38,433 --> 03:43:41,736 LOTS OF BARRIERS TO CLINICAL 6079 03:43:41,736 --> 03:43:43,805 TRIAL PARTICIPATION FOR PEOPLE, 6080 03:43:43,805 --> 03:43:46,541 BE THEY DISTANCE, KNOWLEDGE, 6081 03:43:46,541 --> 03:43:49,410 ACCESS, INCLUSION EXCLUSION 6082 03:43:49,410 --> 03:43:51,012 CRITERIA, A SUPPORT PERSON TO BE 6083 03:43:51,012 --> 03:43:52,079 WITH THEM DURING THEIR VISITS 6084 03:43:52,079 --> 03:43:59,987 AND SO ON, AND SO, WE RECOGNIZE 6085 03:43:59,987 --> 03:44:01,656 THAT INPUTS FROM THOSE PATIENTS 6086 03:44:01,656 --> 03:44:02,590 OR CAREGIVERS AS TRIALS ARE 6087 03:44:02,590 --> 03:44:03,991 DEVELOPED AND ALONG THE WAY IS 6088 03:44:03,991 --> 03:44:07,728 REALLY IMPORTANT AND VITAL AND I 6089 03:44:07,728 --> 03:44:11,599 THINK THIS IS REFLECTED NICELY 6090 03:44:11,599 --> 03:44:14,936 IN DR. Mc CORMAC'S CONFERENCE, 6091 03:44:14,936 --> 03:44:16,103 WHAT'S IMPORTANT TO THEM IN 6092 03:44:16,103 --> 03:44:18,873 GETTING THAT FEEDBACK WE'RE 6093 03:44:18,873 --> 03:44:19,874 PLANNING. 6094 03:44:19,874 --> 03:44:25,179 AND I THINKAN THA IS MY LAST 6095 03:44:25,179 --> 03:44:25,379 SLIDE. 6096 03:44:25,379 --> 03:44:28,015 I THINK IT'S TIME WE CAN GO TO 6097 03:44:28,015 --> 03:44:28,983 OUR PANEL. 6098 03:44:28,983 --> 03:44:31,519 AS I REMINDER YOU CAN POST YOUR 6099 03:44:31,519 --> 03:44:36,657 QUESTIONS IN THE Q&A TOOL. 6100 03:44:36,657 --> 03:44:37,091 ON THE SCREEN. 6101 03:44:37,091 --> 03:44:40,328 AND WE WILL BRING OUR PANELISTS 6102 03:44:40,328 --> 03:44:40,528 BACK. 6103 03:44:40,528 --> 03:44:41,863 >> JUST GIVE US A SECOND TO 6104 03:44:41,863 --> 03:44:52,373 BRING YOU INTO PANELISTS MODE. 6105 03:44:55,776 --> 03:45:01,082 DR. NETWORK ICK E-- NICKERSON, 6106 03:45:01,082 --> 03:45:03,184 CAN YOU TURN ON YOUR CAMERA AND 6107 03:45:03,184 --> 03:45:04,952 JOIN US, ALL RIGHT, I THINK WE 6108 03:45:04,952 --> 03:45:05,820 HAVE EVERYBODY. 6109 03:45:05,820 --> 03:45:06,254 >> GREAT. 6110 03:45:06,254 --> 03:45:06,888 >> WHILE WE'RE WAITING FOR 6111 03:45:06,888 --> 03:45:09,290 QUESTIONS TO COME INTO THE TOOL, 6112 03:45:09,290 --> 03:45:13,127 YOU KNOW I THINK THERE WAS A 6113 03:45:13,127 --> 03:45:14,862 COMMON THEME OF REALLY HAVING, 6114 03:45:14,862 --> 03:45:17,665 YOU KNOW A LOT OF DIFFERENT 6115 03:45:17,665 --> 03:45:19,433 BARRIERS TO COMPLETING CLINICAL 6116 03:45:19,433 --> 03:45:21,969 TRIALS, ENROLLING IN CLINICAL 6117 03:45:21,969 --> 03:45:24,472 TRIALS, AND I JUST WANTED TO 6118 03:45:24,472 --> 03:45:25,139 TOUCH ON THAT. 6119 03:45:25,139 --> 03:45:26,807 I THINK WITH A COUPLE OF 6120 03:45:26,807 --> 03:45:28,643 QUESTIONS AND I WILL JUST START 6121 03:45:28,643 --> 03:45:31,379 WITH 1 UNTIL WE CAN GELT 6122 03:45:31,379 --> 03:45:34,115 QUESTIONS COMING IN, BUT I WILL 6123 03:45:34,115 --> 03:45:35,550 FEED ON THIS THEME IF NEEDED. 6124 03:45:35,550 --> 03:45:41,122 SO YOU KNOW WITH THE ERA OF 6125 03:45:41,122 --> 03:45:42,890 COVID, A KNOW A LOT OF CLINICAL 6126 03:45:42,890 --> 03:45:44,859 STUDIES HAD TO PAUSE BECAUSE WE 6127 03:45:44,859 --> 03:45:48,796 COULDN'T CONDUCT RESEARCH VISITS 6128 03:45:48,796 --> 03:45:49,530 RELATED TO OBSERVATIONAL STUDIES 6129 03:45:49,530 --> 03:45:51,198 AND SOMETIMES WE WERE HAVING 6130 03:45:51,198 --> 03:45:51,766 DIFFICULTIES WITH CLINICAL 6131 03:45:51,766 --> 03:45:53,100 TRIALS AND THERE WAS A MOVEMENT, 6132 03:45:53,100 --> 03:45:55,503 I KNOW BY A LOT OF GROUPS, 6133 03:45:55,503 --> 03:45:57,238 SPECIALLY IN THE PULMONARY AND 6134 03:45:57,238 --> 03:45:59,607 SLEEP RESEARCH COMMUNITY TO MOVE 6135 03:45:59,607 --> 03:46:02,143 INTO YOU KNOW REMOTE ACCESS TO 6136 03:46:02,143 --> 03:46:04,645 PATIENTS AND THEN IN TURN, THINK 6137 03:46:04,645 --> 03:46:06,280 ABOUT NOVEL WAYS TO CONDUCT 6138 03:46:06,280 --> 03:46:07,448 CLINICAL TRIALS AND I THINK THAT 6139 03:46:07,448 --> 03:46:08,816 THIS IS AN IMPORTANT THING THAT 6140 03:46:08,816 --> 03:46:13,354 MAYBE YOU ALL COULD DISCUSS 6141 03:46:13,354 --> 03:46:14,956 BECAUSE IT RELATES BACK TO WHAT 6142 03:46:14,956 --> 03:46:20,628 END POINTS THE FDA MIGHT ACCEPT. 6143 03:46:20,628 --> 03:46:21,829 AS, YOU KNOW TRIALS ARE GOING 6144 03:46:21,829 --> 03:46:24,031 INTO THE FUTURE AND THE IDEA OF 6145 03:46:24,031 --> 03:46:26,567 EQUITABLE ACCESS, IN REACHING 6146 03:46:26,567 --> 03:46:29,103 OUTSIDE OF THOSE HIGH RESOURCE 6147 03:46:29,103 --> 03:46:30,938 ACADEMIC RESEARCH CENTERS, SO, I 6148 03:46:30,938 --> 03:46:32,473 THINK THAT ANYBODY CAN START 6149 03:46:32,473 --> 03:46:34,408 WITH THAT QUESTION, BUT I THINK 6150 03:46:34,408 --> 03:46:35,710 THAT THAT'S AN IMPORTANT THING 6151 03:46:35,710 --> 03:46:37,111 THAT WE SHOULD TALK ABOUT IN 6152 03:46:37,111 --> 03:46:39,146 REALLY THINKING ABOUT THE 6153 03:46:39,146 --> 03:46:41,048 BARRIERS AND FACILITATORS TO 6154 03:46:41,048 --> 03:46:47,121 ACCESS AND ENROLLMENT FOR THESE 6155 03:46:47,121 --> 03:46:48,422 CLINICAL TRIALS. 6156 03:46:48,422 --> 03:46:49,924 >> I CAN START. 6157 03:46:49,924 --> 03:46:52,293 HOPEFULLY THAT WILL -- MY 6158 03:46:52,293 --> 03:46:54,128 COMMENTS WILL HELP PROMPT SOME 6159 03:46:54,128 --> 03:46:55,429 ADDITIONAL DISCUSSION BUT THIS 6160 03:46:55,429 --> 03:46:58,232 IS AN ONGOING DISCUSSION AMONG 6161 03:46:58,232 --> 03:47:01,135 OUR -- WE HAVE A WORKING GROUP 6162 03:47:01,135 --> 03:47:02,870 THROUGH OUR CARE CENTER NETWORK 6163 03:47:02,870 --> 03:47:07,575 FOR RURAL HEALTH ACCESS AND SO 6164 03:47:07,575 --> 03:47:09,644 WE UNDERSTAND THAT NOT ONLY 6165 03:47:09,644 --> 03:47:11,078 ACCESS TO CLINICAL CARE BUT 6166 03:47:11,078 --> 03:47:12,246 CLINICAL TRIAL VERY LIMITED FOR 6167 03:47:12,246 --> 03:47:14,582 PATIENTS IN THE RURAL SPACE. 6168 03:47:14,582 --> 03:47:16,584 CERTAINLY THERE ARE BARRIERS TO 6169 03:47:16,584 --> 03:47:18,085 ACCESS AND MORE SUBURBAN AND 6170 03:47:18,085 --> 03:47:24,258 URBAN AREAS AS WELL, BUT 6171 03:47:24,258 --> 03:47:25,493 DISTANCE, TRAVEL, TIME SPENT, 6172 03:47:25,493 --> 03:47:26,527 THINGS LIKE THAT, PARTICULARLY 6173 03:47:26,527 --> 03:47:28,496 FOR PATIENTS WHO NEED A 6174 03:47:28,496 --> 03:47:30,865 CAREGIVER, WHO ARE ON OXYGEN, 6175 03:47:30,865 --> 03:47:31,899 THEY HAVE LIMITATIONS IN HOW 6176 03:47:31,899 --> 03:47:34,168 LONG THEY CAN BE AWAY FROM HOME. 6177 03:47:34,168 --> 03:47:35,836 I SEE HEADS NODDING, ARE 6178 03:47:35,836 --> 03:47:40,274 BARRIERS AND I THINK WE HAVE -- 6179 03:47:40,274 --> 03:47:43,778 THERE ARE IDENTIFIED SOME 6180 03:47:43,778 --> 03:47:46,180 SUCCESSFUL EXAMPLES, MORE PROOF 6181 03:47:46,180 --> 03:47:50,317 OF CONCEPT, I THINK IN 6182 03:47:50,317 --> 03:47:52,586 DECENTRALIZED TRIALS IN 6183 03:47:52,586 --> 03:47:56,424 PULMONARY FIBROSIS, WHERE THE 6184 03:47:56,424 --> 03:47:59,794 PATIENTS CAN ENROLL VIRTUALLY 6185 03:47:59,794 --> 03:48:02,096 AND THEN DO STUDY ASSESSMENTS 6186 03:48:02,096 --> 03:48:06,934 LOCALLY AND HAVE THEM SENT TO A 6187 03:48:06,934 --> 03:48:07,868 CENTRAL MONITOR. 6188 03:48:07,868 --> 03:48:12,573 THERE'S RECENTLY A STUDY OF 6189 03:48:12,573 --> 03:48:14,175 NANTIDINIB IN MY O 6190 03:48:14,175 --> 03:48:15,443 [INDISCERNIBLE] ILD, THE 6191 03:48:15,443 --> 03:48:16,277 UNIVERSITY OF COLORADO'S APPLE 6192 03:48:16,277 --> 03:48:19,246 STUDY DID A LOT OF REMOTE DATA 6193 03:48:19,246 --> 03:48:21,315 COLLECTION FOR THEIR FAMILIAL 6194 03:48:21,315 --> 03:48:26,987 COHORT THERE, SO I THINK THAT 6195 03:48:26,987 --> 03:48:29,857 IT'S UNDERSTANDING WHAT THOSE 6196 03:48:29,857 --> 03:48:32,793 CHALLENGES ARE BECAUSE FOR THE 6197 03:48:32,793 --> 03:48:33,661 MORE REGISTRATIONAL STUDIES THE 6198 03:48:33,661 --> 03:48:35,963 DATA HAS TO BE CLEAN AND I THINK 6199 03:48:35,963 --> 03:48:37,264 THE CHALLENGES WITH THOSE LOCAL 6200 03:48:37,264 --> 03:48:39,366 COLLECTIONS OF DATA WAS THE 6201 03:48:39,366 --> 03:48:42,336 DIFFERENCES IN PERHAPS CT SCANS 6202 03:48:42,336 --> 03:48:43,537 OR PFT MEASURE AMS AND THINGS 6203 03:48:43,537 --> 03:48:48,209 LIKE THAT, ACTUAL ACTUALLY COM, 6204 03:48:48,209 --> 03:48:50,044 IT WASN'T THE PATIENT'S 6205 03:48:50,044 --> 03:48:50,978 PARTICIPATION, THEY WERE WILLING 6206 03:48:50,978 --> 03:48:53,047 TO DO THOSE THINGS BUT THE 6207 03:48:53,047 --> 03:48:54,148 FIDELITY OF THE DATA THEY WERE 6208 03:48:54,148 --> 03:48:55,282 GETTING OUT OF THAT. 6209 03:48:55,282 --> 03:48:56,717 I WOULD LOVE TO HEAR THE 6210 03:48:56,717 --> 03:49:00,988 EXPERIENCE IN OTHER AREAS ON 6211 03:49:00,988 --> 03:49:01,188 THAT. 6212 03:49:01,188 --> 03:49:02,723 >> WE'VE DONE A LITTLE BIT WITH 6213 03:49:02,723 --> 03:49:04,992 THAT, AND PART OF THE ISSUE THAT 6214 03:49:04,992 --> 03:49:07,094 WE'VE RUN INTO WITH REMOTE 6215 03:49:07,094 --> 03:49:08,629 TRIALS IS VALIDATION OF THE 6216 03:49:08,629 --> 03:49:10,030 TOOLS THAT ARE -- ARK 6217 03:49:10,030 --> 03:49:10,965 ASSESSESMENT TOOLS THAT ARE 6218 03:49:10,965 --> 03:49:15,202 BEING USED SO FOR INSTANCE, 6219 03:49:15,202 --> 03:49:16,604 COUGH CONING AT NIGHT, IT SOUNDS 6220 03:49:16,604 --> 03:49:18,439 GREAT BUT IF YOU DON'T SLEEP BY 6221 03:49:18,439 --> 03:49:23,310 YOURSELF IN A QUIET ROOM, IT'S 6222 03:49:23,310 --> 03:49:29,850 NOT RELIABLE, IF YOUR DOG 6223 03:49:29,850 --> 03:49:32,219 COUGHS, IT'S NOT RELIABLE, SO 6224 03:49:32,219 --> 03:49:33,187 WE'RE WORKING WITH SOME AGENCIES 6225 03:49:33,187 --> 03:49:36,323 TO SEE IF THIS PASSIVE DATA CAN 6226 03:49:36,323 --> 03:49:39,794 BE VALIDATED FOR COLLECTION BUT 6227 03:49:39,794 --> 03:49:41,629 I THINK WELL'S SOME SKEPTICISM 6228 03:49:41,629 --> 03:49:48,235 UNTIL WE CAN GET VALIDATION OF 6229 03:49:48,235 --> 03:49:53,407 SPIRITUAL STRUGGLES 6230 03:49:53,407 --> 03:49:53,974 O--METABOLIZEDETRY, REMOTE 6231 03:49:53,974 --> 03:49:54,308 SPIROMETRY. 6232 03:49:54,308 --> 03:49:56,110 >> I WOULD ADD THERE'S 2 ANGLES 6233 03:49:56,110 --> 03:49:57,778 TO THE QUESTION, RIGHT WHICH IS 6234 03:49:57,778 --> 03:50:00,147 1 THE USE OF -- THE VALIDATION 6235 03:50:00,147 --> 03:50:01,215 OF TECHNOLOGY FOR YOU KNOW 6236 03:50:01,215 --> 03:50:05,419 THERE'S A HIGH DEGREE OF 6237 03:50:05,419 --> 03:50:06,587 SENSITIVITY AND SPECIFICITY AND 6238 03:50:06,587 --> 03:50:07,755 IN PRECISION NEEDED FOR CLINICAL 6239 03:50:07,755 --> 03:50:09,490 TRIAL ANDS THEN THERE'S THE 6240 03:50:09,490 --> 03:50:10,958 QUESTION OF, YOU KNOW WHAT'S 6241 03:50:10,958 --> 03:50:13,360 USEFUL FOR PATIENTS, TOO, AND I 6242 03:50:13,360 --> 03:50:15,129 KNOW THE CF FOUNDATION 6243 03:50:15,129 --> 03:50:20,301 SPONSORED, I RECEIVED A PORTABLE 6244 03:50:20,301 --> 03:50:22,937 SPIROMETRY DEVICE DURING EARLY 6245 03:50:22,937 --> 03:50:25,239 COVID AND FORTUNATELY, COVID 6246 03:50:25,239 --> 03:50:27,875 ALSO COINCIDED WITH TRIKAFTA'S 6247 03:50:27,875 --> 03:50:29,109 APPROVAL SO FOR MANY OF US IT 6248 03:50:29,109 --> 03:50:30,344 WAS A VERY DIFFERENT TIME BUT 6249 03:50:30,344 --> 03:50:31,946 THE POINT I THINK IS IN RECENT 6250 03:50:31,946 --> 03:50:34,782 YEARS, I THINK THERE'S BEEN SOME 6251 03:50:34,782 --> 03:50:36,917 INVESTIGATION INTO THE 6252 03:50:36,917 --> 03:50:38,419 VALIDATION AND COMPARISON OF A 6253 03:50:38,419 --> 03:50:41,455 LOT OF THESE PORTABLE SPIROMETRY 6254 03:50:41,455 --> 03:50:42,723 DEVICES BUT AT THE END OF THE 6255 03:50:42,723 --> 03:50:43,691 DAY WHAT'S USEFUL TO PATIENTS A 6256 03:50:43,691 --> 03:50:45,659 LOT OF TIME SYSTEM NOT 6257 03:50:45,659 --> 03:50:47,061 NECESSARILY THE ABSOLUTE VALUE 6258 03:50:47,061 --> 03:50:48,596 BUT DAY-TO-DAY CHANGES AND 6259 03:50:48,596 --> 03:50:49,797 MEASURING AND SEEING AND REALLY 6260 03:50:49,797 --> 03:50:51,198 SEEING THE PATTERN AND THE 6261 03:50:51,198 --> 03:50:51,532 TREND. 6262 03:50:51,532 --> 03:50:53,000 AND SO, I WOULD LIKE TO THINK, 6263 03:50:53,000 --> 03:50:54,835 YOU KNOW COVID WAS HORRIBLE FOR 6264 03:50:54,835 --> 03:50:55,903 A MILLION DIFFERENT REASONS BUT 6265 03:50:55,903 --> 03:50:58,072 I THINK THAT IT ALSO OPENED US 6266 03:50:58,072 --> 03:50:59,506 TO YOU KNOW THE POINTS THAT 6267 03:50:59,506 --> 03:51:01,108 MICHELLE AND AMY HAVE MADE THAT 6268 03:51:01,108 --> 03:51:03,043 IT WAS AN OPPORTUNITY TO 6269 03:51:03,043 --> 03:51:07,514 EXPERIMENT AND SEE HOW WE CAN 6270 03:51:07,514 --> 03:51:10,618 ADDRESS THE STILL MASSIVE 6271 03:51:10,618 --> 03:51:13,587 DISPARAGING DISEASES IN 6272 03:51:13,587 --> 03:51:14,488 COMPLIEWNTS IMEGGABLE ESPECIALLY 6273 03:51:14,488 --> 03:51:16,390 IN RURAL AND MINORITIZED GROUPS. 6274 03:51:16,390 --> 03:51:17,791 SO IT'S IMPORTANT TO USE 6275 03:51:17,791 --> 03:51:18,726 OPPORTUNITIES LIKE THAT TO 6276 03:51:18,726 --> 03:51:20,361 INNOVATE AND BE A BIT MORE 6277 03:51:20,361 --> 03:51:24,932 WILLING TO BE DYNAMIC WITH, YOU 6278 03:51:24,932 --> 03:51:25,966 KNOW THE SYSTEM. 6279 03:51:25,966 --> 03:51:29,737 >> I WOULD -- I COMPLETELY AGREE 6280 03:51:29,737 --> 03:51:32,473 THAT THE ACCURACY OF PULMONARY 6281 03:51:32,473 --> 03:51:34,308 FUNCTION TESTS IS PARAMOUNT IN 6282 03:51:34,308 --> 03:51:36,076 SOME OF THESE TRIALS AND THAT 6283 03:51:36,076 --> 03:51:37,244 THERE ARE END POINTS THAT ARE 6284 03:51:37,244 --> 03:51:39,213 MORE IMPORTANT THAN OTHERS IN 6285 03:51:39,213 --> 03:51:40,481 THIS 2 YEAR LONG MILD TRIAL. 6286 03:51:40,481 --> 03:51:43,150 WE WANT TO BE SURE THAT PATIENTS 6287 03:51:43,150 --> 03:51:44,685 HAVE THEIR PULMONARY FUNCTION 6288 03:51:44,685 --> 03:51:45,986 TESTS AT OUR LABORATORY AND 6289 03:51:45,986 --> 03:51:50,057 WE'VE RELAXED THE NEED TO HAVE 6290 03:51:50,057 --> 03:51:51,458 PULMONARY FUNCTION TESTS YOU 6291 03:51:51,458 --> 03:51:52,993 KNOW AT OUR LABORATORY FOR 6292 03:51:52,993 --> 03:51:53,961 REMOTE VISITS THAT OCCUR IN 6293 03:51:53,961 --> 03:51:54,194 BETWEEN. 6294 03:51:54,194 --> 03:51:55,262 AND I THINK THAT'S GOING TO 6295 03:51:55,262 --> 03:51:56,697 BECOME A PART OF OUR TRIALS 6296 03:51:56,697 --> 03:51:58,232 GOING FORWARD, I THINK THERE'S 6297 03:51:58,232 --> 03:52:01,435 DEFINITELY A PLACE FOR REMOTE 6298 03:52:01,435 --> 03:52:01,669 VISITS. 6299 03:52:01,669 --> 03:52:03,170 WE WILL PROBABLY DO A HYBRID 6300 03:52:03,170 --> 03:52:05,406 MODEL WHERE WE HAVE BOTH REMOTE 6301 03:52:05,406 --> 03:52:06,473 AND IN-PERSON VISITS AT THE SITE 6302 03:52:06,473 --> 03:52:08,008 SO WE HAVE AT LEAST SOME 6303 03:52:08,008 --> 03:52:10,711 MEASURES THAT ARE KNOWN TO BE 6304 03:52:10,711 --> 03:52:16,116 DONE AND CALIBRATED, YOU KNOW 6305 03:52:16,116 --> 03:52:18,852 HIGH QUALITY ATS, WELL DRIVEN 8 6306 03:52:18,852 --> 03:52:20,254 PULMONARY FUNCTION LABS AND IN 6307 03:52:20,254 --> 03:52:24,458 THE CONTEXT OF MILD, DOING A 6308 03:52:24,458 --> 03:52:25,659 PARALLEL HOME SPIROMETRY STUDY 6309 03:52:25,659 --> 03:52:27,828 LIKE TRE WAS TALKING ABOUT AND 6310 03:52:27,828 --> 03:52:29,563 WE'VE SEEN VERY GOOD TREND 6311 03:52:29,563 --> 03:52:31,765 AGREEMENT WITH WHAT'S FOUND WITH 6312 03:52:31,765 --> 03:52:33,534 HOME SPIROMETRY AND OFFICE 6313 03:52:33,534 --> 03:52:34,234 SPIROMETRY. 6314 03:52:34,234 --> 03:52:35,736 THE ABSOLUTE VALUES ARE NOT 6315 03:52:35,736 --> 03:52:36,837 IDENTICAL BUT THE TRENDS ARE 6316 03:52:36,837 --> 03:52:39,273 OVERLAPPING AND I THINK THAT CAN 6317 03:52:39,273 --> 03:52:40,541 BECOME AN END POINT IN THE 6318 03:52:40,541 --> 03:52:44,478 FUTURE FOR SOME STUDIES. 6319 03:52:44,478 --> 03:52:45,980 >> AND STUDY SELECTION WOULD BE 6320 03:52:45,980 --> 03:52:46,780 IMPORTANT TOO BECAUSE SOME 6321 03:52:46,780 --> 03:52:50,584 STUDIES MAY BE MORE AMENABLE TO 6322 03:52:50,584 --> 03:52:51,852 REMOTE PARTICIPATION AND EVEN IF 6323 03:52:51,852 --> 03:52:55,289 WE COULD DO AS MUCH REMOTELY AS 6324 03:52:55,289 --> 03:52:56,824 POSSIBLE, THAT STILL WOULD LIMIT 6325 03:52:56,824 --> 03:53:03,831 THE BURDEN ON PATIENTS TO DO 6326 03:53:03,831 --> 03:53:04,231 IN-PERSON VISITS. 6327 03:53:04,231 --> 03:53:06,033 >> YEAH, I THINK THOSE ARE ALL 6328 03:53:06,033 --> 03:53:07,368 GOOD POINTS AS WAYS FOR US TO 6329 03:53:07,368 --> 03:53:09,003 SORT OF TRY TO REDUCE THE 6330 03:53:09,003 --> 03:53:12,206 BARRIERS AND THEN OF COURSE, THE 6331 03:53:12,206 --> 03:53:13,640 LIMITATIONS ACROSS RARE DISEASES 6332 03:53:13,640 --> 03:53:15,809 IS ACCESS TO NUMBERS OF 6333 03:53:15,809 --> 03:53:16,777 PARTICIPANTS AND THE MORE WAYS 6334 03:53:16,777 --> 03:53:19,346 THAT THE STUDIES CAN BE DESIGNED 6335 03:53:19,346 --> 03:53:20,748 TO INCREASE ACCESS WOULD MAKE 6336 03:53:20,748 --> 03:53:25,052 THEM MORE SUCCESSFUL AS WELL. 6337 03:53:25,052 --> 03:53:26,653 >> WE DO HAVE 1 QUESTION IN THE 6338 03:53:26,653 --> 03:53:28,222 Q&A TOOL AND I INVITE OTHER 6339 03:53:28,222 --> 03:53:30,190 MEMBERS OF THE AUDIENCE TO POSE 6340 03:53:30,190 --> 03:53:31,425 QUESTIONS DURING THE DISCUSSION. 6341 03:53:31,425 --> 03:53:33,560 SO THIS 1 IS CENTER DR. MOFFIT, 6342 03:53:33,560 --> 03:53:35,662 IT SAYS WE'VE BEEN HEARING THAT 6343 03:53:35,662 --> 03:53:37,865 PATIENTS ARE EXCITED ABOUT 6344 03:53:37,865 --> 03:53:40,601 INNOVATIVE THERAPY BUT ONLY ONCE 6345 03:53:40,601 --> 03:53:40,968 APPROVED. 6346 03:53:40,968 --> 03:53:43,270 THEY'RE RELUCTANT TO PARTICIPATE 6347 03:53:43,270 --> 03:53:46,173 IN CLINICAL TRIALS GIVEN THE 6348 03:53:46,173 --> 03:53:46,807 UNCERTAINTY OF NOVEL THERAPIES 6349 03:53:46,807 --> 03:53:47,775 AND WHAT ARE THINGS THAT 6350 03:53:47,775 --> 03:53:49,843 INDUSTRY CAN DO TO BREAK DOWN 6351 03:53:49,843 --> 03:53:51,979 THESE BARRIER ANDS COMMUNICATE 6352 03:53:51,979 --> 03:53:53,547 MORE EFFECTIVELY TO PATIENTS 6353 03:53:53,547 --> 03:53:54,314 ABOUT THESE OPPORTUNITIES. 6354 03:53:54,314 --> 03:53:56,116 AND BEFORE WE GO THERE, I WILL 6355 03:53:56,116 --> 03:53:58,819 ADD TO THAT, IN REALLY THINKING 6356 03:53:58,819 --> 03:53:59,987 ABOUT COMPETITION BETWEEN MOFLE 6357 03:53:59,987 --> 03:54:01,722 THERAPIES AND IN THE GENE 6358 03:54:01,722 --> 03:54:03,557 THERAPY SPACE, YOU KNOW, THIS 6359 03:54:03,557 --> 03:54:06,960 MAY BE A PRIME ISSUE, AT THIS 6360 03:54:06,960 --> 03:54:09,063 TIME, BUT REALLY THINKING ABOUT 6361 03:54:09,063 --> 03:54:09,997 THE OTHER ALTERNATIVE 6362 03:54:09,997 --> 03:54:11,131 INTERVENTIONS THAT MAY COME IN 6363 03:54:11,131 --> 03:54:14,601 THE NEXT 5 YEARS, HOW WILL 6364 03:54:14,601 --> 03:54:16,770 INDIVIDUALS NEED TO BE EDUCATED 6365 03:54:16,770 --> 03:54:18,038 ABOUT THESE OPPORTUNITIES TO 6366 03:54:18,038 --> 03:54:20,007 HELP MAKE THESE CHOICES. 6367 03:54:20,007 --> 03:54:21,408 AND MAYBE, TRE, I WILL PICK ON 6368 03:54:21,408 --> 03:54:24,511 YOU FIRST TO ANSWER THIS 6369 03:54:24,511 --> 03:54:25,045 QUESTION. 6370 03:54:25,045 --> 03:54:26,380 >> YEAH, I WILL -- YEAH, I MEAN 6371 03:54:26,380 --> 03:54:28,382 I THINK IT'S AN INCREDIBLY 6372 03:54:28,382 --> 03:54:29,183 IMPORTANT AND SENSITIVE TOPIC 6373 03:54:29,183 --> 03:54:32,786 RIGHT NOW AND I THINK IT'S -- 1 6374 03:54:32,786 --> 03:54:34,288 OF THE -- 1 OF MY FAVORITE 6375 03:54:34,288 --> 03:54:36,890 CLASSES IS IN MY UNDERGRAD WAS 6376 03:54:36,890 --> 03:54:38,292 LEARNING ABOUT THE SORTING 6377 03:54:38,292 --> 03:54:40,394 SIGNALS OF SEE SAW OF PATIENT 6378 03:54:40,394 --> 03:54:41,161 AUTONOMY AND PHYSICIAN 6379 03:54:41,161 --> 03:54:42,563 PATERNALISM AND I THINK THAT'S 6380 03:54:42,563 --> 03:54:44,198 VERY MUCH WHAT WE'RE TALKING 6381 03:54:44,198 --> 03:54:50,704 ABOUT HERE, ESPECIALLY WITH 6382 03:54:50,704 --> 03:54:51,905 NOVEL INNOVATIVE THERAPIES, IN 6383 03:54:51,905 --> 03:54:53,307 THE GENE THERAPY SPACE 6384 03:54:53,307 --> 03:54:54,241 ESPECIALLY WHEN DISCUSSING 6385 03:54:54,241 --> 03:54:55,909 VACCINES, I THINK THERE'S A LOT 6386 03:54:55,909 --> 03:54:57,111 OF CONFUSION ABOUT A LOT OF 6387 03:54:57,111 --> 03:54:57,945 THINGS AND ALSO ACKNOWLEDGE THAT 6388 03:54:57,945 --> 03:54:59,546 I THINK WE HAVE AN INFORMATION 6389 03:54:59,546 --> 03:55:00,948 ENVIRONMENT THAT POSES A LOT OF 6390 03:55:00,948 --> 03:55:02,149 CHALLENGES FOR PEOPLE AND 6391 03:55:02,149 --> 03:55:03,650 CONSUMERS AND I OFTEN THINK IT'S 6392 03:55:03,650 --> 03:55:04,618 KIND OF INTERESTING THAT I'M A 6393 03:55:04,618 --> 03:55:06,787 PATIENT IN A LOT OF WAYS BUT I'M 6394 03:55:06,787 --> 03:55:10,224 ALSO JUST A PERSON WHO YOU KNOW, 6395 03:55:10,224 --> 03:55:13,193 WE ALL COULD BE A PATIENT AT ANY 6396 03:55:13,193 --> 03:55:14,128 GIVEN CIRCUMSTANCE, RIGHT, WE'RE 6397 03:55:14,128 --> 03:55:16,196 ALL A PATIENT WHEN WE GET URGENT 6398 03:55:16,196 --> 03:55:17,064 CARE AND 1 THING THAT'S 6399 03:55:17,064 --> 03:55:18,265 IMPORTANT IS THAT WE EMPHASIZE 6400 03:55:18,265 --> 03:55:20,434 THE NEED FOR CLINICIANS TO TALK 6401 03:55:20,434 --> 03:55:23,604 TO PATIENTS LIKE THEIR PEOPLE 6402 03:55:23,604 --> 03:55:25,572 AND FEEL CONFIDENT THAT YOU CAN 6403 03:55:25,572 --> 03:55:27,441 TALK TO HUMANS AND EMPOWER THEM 6404 03:55:27,441 --> 03:55:30,978 IN A WAY THAT DOESN'T BRIDGE TOO 6405 03:55:30,978 --> 03:55:32,513 CLOSE TO PATERNALISM WHILE ALSO 6406 03:55:32,513 --> 03:55:33,147 RECOGNIZING THAT IT'S REALLY 6407 03:55:33,147 --> 03:55:35,649 IMPORTANT TO SPEAK IN A HEALTH 6408 03:55:35,649 --> 03:55:36,583 LITERAL MANNER, THAT'S SOMETHING 6409 03:55:36,583 --> 03:55:38,585 WE TALKING A LOT ABOUT AT THE 6410 03:55:38,585 --> 03:55:40,921 FNIH AND MAKING SURE WE'RE 6411 03:55:40,921 --> 03:55:41,955 COMMUNICATING THESE DEEPLY 6412 03:55:41,955 --> 03:55:43,023 DIFFICULT -- SOME OF THE 6413 03:55:43,023 --> 03:55:44,458 PROJECTS WE HAVE ARE DIFFICULT 6414 03:55:44,458 --> 03:55:48,195 TO CONVEY TO THE CLINICIANS IN 6415 03:55:48,195 --> 03:55:49,596 THE FIELD SO LET ALONE THE 6416 03:55:49,596 --> 03:55:50,797 PHYSICIANS IN THE FIELD. 6417 03:55:50,797 --> 03:55:52,666 SO THAT'S WHERE IT'S REALLY 6418 03:55:52,666 --> 03:55:54,168 INCUMBENT ON FOLKS WHO ARE ISHT 6419 03:55:54,168 --> 03:55:56,036 ACTING WITH PATIENTS A LOT TO 6420 03:55:56,036 --> 03:55:58,105 CONTINUE NURTURING THAT SKILL OF 6421 03:55:58,105 --> 03:55:58,805 HEALTH LITERATURE COMMUNICATIONS 6422 03:55:58,805 --> 03:56:01,909 IN A WAY THAT ENABLES PATIENTS 6423 03:56:01,909 --> 03:56:03,076 TO UNDERSTAND WITHOUT FEELING 6424 03:56:03,076 --> 03:56:06,046 LIKE PEOPLE ARE BEING DICTATED 6425 03:56:06,046 --> 03:56:06,513 TOO. 6426 03:56:06,513 --> 03:56:07,881 I ALMOST MENTIONED THIS DURING 6427 03:56:07,881 --> 03:56:09,783 MY TALK BUT I HAD TO CUT IT FOR 6428 03:56:09,783 --> 03:56:10,851 TIME BUT THERE'S BEEN THE TALK 6429 03:56:10,851 --> 03:56:12,519 ABOUT THE TERM IN THE PAST USED 6430 03:56:12,519 --> 03:56:14,354 TO BE COMPLIANCE WITH PEOPLE 6431 03:56:14,354 --> 03:56:16,523 TAKING MEDICATIONS AND I THINK 6432 03:56:16,523 --> 03:56:18,292 WE'VE SORT OF SHIFTED GENERALLY 6433 03:56:18,292 --> 03:56:19,359 CLOSER TO ADHERENCE AND I THINK 6434 03:56:19,359 --> 03:56:22,329 WE TRY TO STRAY AWAY FROM ANY OF 6435 03:56:22,329 --> 03:56:24,298 THIS TERMINOLOGY IN GENERAL AND 6436 03:56:24,298 --> 03:56:25,399 COMPLIANCE SUGGESTERSA A POWER 6437 03:56:25,399 --> 03:56:26,433 DYNAMIC BETWEEN THE PATIENT AND 6438 03:56:26,433 --> 03:56:28,936 HELT CARE SYSTEM AND I THINK 6439 03:56:28,936 --> 03:56:30,070 THAT DOESN'T REALLY UNDERSTAND 6440 03:56:30,070 --> 03:56:31,705 HOW MUSEUM MANS BEHAVE AND I 6441 03:56:31,705 --> 03:56:33,240 THINK WHEN YOU TURN AND START TO 6442 03:56:33,240 --> 03:56:35,876 USE THE WORD COMPLIANCE, YOU 6443 03:56:35,876 --> 03:56:37,544 START TO SHIFT, SHIFT WHAT 6444 03:56:37,544 --> 03:56:38,278 ACTUALLY MOTIVATES HUMANS AND I 6445 03:56:38,278 --> 03:56:40,747 THINK IN THE SAME WAY, 6446 03:56:40,747 --> 03:56:42,115 COMMUNICATING ABOUT NOVEL 6447 03:56:42,115 --> 03:56:42,950 THERAPIES IS REALLY ABOUT 6448 03:56:42,950 --> 03:56:47,421 UNDERSTANDING THAT THERE HAS TO 6449 03:56:47,421 --> 03:56:48,922 BE AGENCY INVOLVED WHEN PATIENTS 6450 03:56:48,922 --> 03:56:50,991 ARE TRYING TO CONSIDER A 6451 03:56:50,991 --> 03:56:52,059 CLINICAL TRIAL AND ALSO 1 OTHER 6452 03:56:52,059 --> 03:56:53,460 POINT I WILL MAKE IS THAT IT'S 6453 03:56:53,460 --> 03:56:56,697 GOT TO BE FRAMED IN THE CONTEXT 6454 03:56:56,697 --> 03:56:58,765 OF MULTIPLE INTERACTIONS, RIGHT? 6455 03:56:58,765 --> 03:57:00,934 YOU'RE NOT REALLY ENROLLING A 6456 03:57:00,934 --> 03:57:03,036 PATIENT, THE DAY YOU ASK THEM TO 6457 03:57:03,036 --> 03:57:05,038 ENROLL IN A CLINICAL TRIAL, YOU 6458 03:57:05,038 --> 03:57:07,040 ARE REALLY ENROLLING THEM IN THE 6459 03:57:07,040 --> 03:57:08,575 WEEKS TO MONTHS TO YEARS DURING 6460 03:57:08,575 --> 03:57:10,844 THAT TRUST BUILDING FOR FUTURE 6461 03:57:10,844 --> 03:57:12,679 CLINICAL TRIALS, SO EVERY 6462 03:57:12,679 --> 03:57:14,414 INTERACTION IS AN OPPORTUNITY TO 6463 03:57:14,414 --> 03:57:16,650 EDUCATE AND INFORM FOLKS. 6464 03:57:16,650 --> 03:57:18,518 >> AND I THINK THAT ADD TO THAT, 6465 03:57:18,518 --> 03:57:21,154 YOU KNOW, I THINK TO JEFF'S 6466 03:57:21,154 --> 03:57:23,557 POINT IS HOW CAN THE -- INDUSTRY 6467 03:57:23,557 --> 03:57:25,158 ALSO BE IN PARTNERS IN THAT, I 6468 03:57:25,158 --> 03:57:27,661 THINK YOU ALLUDED TO THAT, IN 6469 03:57:27,661 --> 03:57:30,464 THE BARRIERS FOR TRANSLATING 6470 03:57:30,464 --> 03:57:32,366 HEALTH INFORMATION IN SOME OF 6471 03:57:32,366 --> 03:57:33,667 THESE NOVEL THERAPYS EVEN TO THE 6472 03:57:33,667 --> 03:57:35,369 CLINICIAN THAT'S GOING TO IN 6473 03:57:35,369 --> 03:57:36,570 TURN HAVE TO TRANSLATE IT BACK 6474 03:57:36,570 --> 03:57:38,272 TO THEIR PATIENT AND I THINK 6475 03:57:38,272 --> 03:57:41,308 THAT THAT IS A ROLE THAT SOME OF 6476 03:57:41,308 --> 03:57:42,442 THE ADVOCACY GROUPS COULD ALSO 6477 03:57:42,442 --> 03:57:44,378 HELP SUPPORT SO MAYBE WE CAN 6478 03:57:44,378 --> 03:57:47,881 HEAR FROM THEIR EXPERIENCE AS 6479 03:57:47,881 --> 03:57:48,315 WELL. 6480 03:57:48,315 --> 03:57:49,650 >> I THINK THERE ALSO REALLY 6481 03:57:49,650 --> 03:57:52,953 NEEDS TO BE CLARITY ABOUT THESE 6482 03:57:52,953 --> 03:57:53,387 NOVEL THERAPIES. 6483 03:57:53,387 --> 03:57:56,023 I KNOW THAT IN OUR GROUP, WE'RE 6484 03:57:56,023 --> 03:57:57,324 TALKING ABOUT MRNA THERAPY AND 6485 03:57:57,324 --> 03:57:59,293 IT IS UNCLEAR EVEN AT THIS 6486 03:57:59,293 --> 03:58:01,628 POINT, IF YOU PARTICIPATE IN 6487 03:58:01,628 --> 03:58:02,929 MRNA THERAPY FOR YOUR GENE, 6488 03:58:02,929 --> 03:58:04,264 RIGHT NOW, AND IT DIDN'T WORK, 6489 03:58:04,264 --> 03:58:06,400 DOES THAT MEAN YOU'RE GOING TO 6490 03:58:06,400 --> 03:58:08,101 BE PRECLUDED FROM THE THERAPIES 6491 03:58:08,101 --> 03:58:09,503 COMING UP IN 5 YEARS AND 6492 03:58:09,503 --> 03:58:10,837 PATIENTS ARE VERY WORRIED. 6493 03:58:10,837 --> 03:58:12,806 IT MAKES THEM WAIT, THEY WANT TO 6494 03:58:12,806 --> 03:58:14,207 WAIT AND SEE IF SOMETHING IS 6495 03:58:14,207 --> 03:58:16,410 GOING TO HELP BEFORE THEY KIND 6496 03:58:16,410 --> 03:58:18,879 OF BLOWN THEIR SHOT. 6497 03:58:18,879 --> 03:58:21,615 SO I THINK INFORMING PATIENTS, 6498 03:58:21,615 --> 03:58:22,349 INFORMED CONSENT. 6499 03:58:22,349 --> 03:58:23,650 MAKING SURE WE ALL UNDERSTAND 6500 03:58:23,650 --> 03:58:23,884 IT. 6501 03:58:23,884 --> 03:58:25,252 I DON'T EVEN KNOW. 6502 03:58:25,252 --> 03:58:26,720 I'VE HEARD 3 DIFFERENT PROPOSALS 6503 03:58:26,720 --> 03:58:28,922 IF ARE THIS, 18 MONTHS, 2 YEARS 6504 03:58:28,922 --> 03:58:30,824 AND 15 YEARS FOR WHEN YOU MIGHT 6505 03:58:30,824 --> 03:58:32,059 BE ABLE TO PARTICIPATE IN 6506 03:58:32,059 --> 03:58:32,993 ANOTHER TRIAL. 6507 03:58:32,993 --> 03:58:35,095 WE NEED MORE CLARITY ON WHAT IS 6508 03:58:35,095 --> 03:58:45,639 REALLY THE CONSEQUENCE S OF IT. 6509 03:58:50,010 --> 03:58:51,545 >> FOR LAM AND PULMONARY 6510 03:58:51,545 --> 03:58:53,613 FIBROSIS WE NOW HAVE FDA 6511 03:58:53,613 --> 03:58:55,015 APPROVED DRUGS AND IT'S 6512 03:58:55,015 --> 03:58:56,750 UNETHICAL TO HAVE PATIENTS 6513 03:58:56,750 --> 03:58:57,451 PARTICIPATE IN AN EXPERIMENT IF 6514 03:58:57,451 --> 03:58:59,353 YOU HAVE A PROVEN THERAPY TO 6515 03:58:59,353 --> 03:59:02,756 OFFER THEM AND IN THOSE DECS WE 6516 03:59:02,756 --> 03:59:04,725 OFTEN HAVE TO WAIT UNTIL 6517 03:59:04,725 --> 03:59:07,527 PATIENTS FAIL THE APPROVED 6518 03:59:07,527 --> 03:59:09,029 THERAPIES, FOR IPF WE HAVE 3 6519 03:59:09,029 --> 03:59:12,399 DRUGS WE CAN CONSIDER AND AT 6520 03:59:12,399 --> 03:59:14,067 THAT POINT, YOU KNOW THEY'RE NOT 6521 03:59:14,067 --> 03:59:15,569 AS -- IT'S NOT THE SAME 6522 03:59:15,569 --> 03:59:17,704 CANDIDATE AT THE END OF 2 FAILED 6523 03:59:17,704 --> 03:59:20,240 THERAPIES OR 3 FAILED THERAPIES 6524 03:59:20,240 --> 03:59:25,579 AND THE TRIAL RESULTS WITH BE 6525 03:59:25,579 --> 03:59:26,146 BIASED BY THAT. 6526 03:59:26,146 --> 03:59:27,414 SO I THINK NOX FAMILY 6527 03:59:27,414 --> 03:59:28,482 ACTIVATORLE THERAPIES IN SOME OF 6528 03:59:28,482 --> 03:59:30,684 THE DOMAINS THAT WE PRACTICE IN 6529 03:59:30,684 --> 03:59:37,023 AREN'T ALL THAT APPEALS TO OUR 6530 03:59:37,023 --> 03:59:37,924 PATIENTS BUT YEAH. 6531 03:59:37,924 --> 03:59:39,860 >> I WAS ACTUALLY GOING TO 6532 03:59:39,860 --> 03:59:41,361 COMMENT ON SOMETHING THAT TRE 6533 03:59:41,361 --> 03:59:47,000 SAID AS WELL AS NOW WHAT 6534 03:59:47,000 --> 03:59:47,601 DR. Mc CORMACK AS BROUGHT UP 6535 03:59:47,601 --> 03:59:50,437 BUT I WILL SAY THE LATTER FIRST, 6536 03:59:50,437 --> 03:59:53,373 I THINK THERE'S A DIFFERENCE 6537 03:59:53,373 --> 03:59:55,675 BETWEEN PATIENTS WHO GET THEIR 6538 03:59:55,675 --> 03:59:57,778 CARE AT CARE CENTERS, RIGHT? 6539 03:59:57,778 --> 04:00:01,281 AND THOSE WHO GET THEIR CARE IN 6540 04:00:01,281 --> 04:00:04,017 A MORE COMMUNITY-BASED PLACE, 6541 04:00:04,017 --> 04:00:04,918 RIGHT? 6542 04:00:04,918 --> 04:00:06,887 THERE ARE LOTS OF BARRIERS TO 6543 04:00:06,887 --> 04:00:10,190 GETTING INTO THOSE CARE CENTERS 6544 04:00:10,190 --> 04:00:13,226 TO START WITH, WAIT TIMES, 6545 04:00:13,226 --> 04:00:18,064 NAVIGATING LARGER SYSTEMS, THE 6546 04:00:18,064 --> 04:00:21,835 DISTANCE AND AND STUFF LIKE THAT 6547 04:00:21,835 --> 04:00:24,371 SO IT HELPS FOR SOME POPULATIONS 6548 04:00:24,371 --> 04:00:25,705 IN SOME WAYS DRIIVE TO BE THERE 6549 04:00:25,705 --> 04:00:27,240 AND I THINK GETTING BACK TO -- 6550 04:00:27,240 --> 04:00:29,776 THAT MAY BE THE CHOIR THAT WE'RE 6551 04:00:29,776 --> 04:00:33,780 ALREADY TALKING TOO MANY TIMES 6552 04:00:33,780 --> 04:00:36,049 IN THOSE CLINICS. 6553 04:00:36,049 --> 04:00:37,350 BUT TAKING A STEP BACK AND 6554 04:00:37,350 --> 04:00:38,485 THINKING ABOUT HOW TREY CAN 6555 04:00:38,485 --> 04:00:41,354 PARTNER WITH THOSE EDUCATIONAL 6556 04:00:41,354 --> 04:00:43,356 OPPORTUNITIES, WE KNOW THAT 1 OF 6557 04:00:43,356 --> 04:00:45,292 THE -- 1 OF THE MOST IMPACTFUL 6558 04:00:45,292 --> 04:00:47,494 THINGS IN TERMS OF ENROLLING IN 6559 04:00:47,494 --> 04:00:48,595 A CLINICAL TRIAL FOR A PATIENT 6560 04:00:48,595 --> 04:00:50,964 IS HAVING A TRUSTED PHYSICIAN 6561 04:00:50,964 --> 04:00:52,466 RECOMMEND THAT FOR THEM. 6562 04:00:52,466 --> 04:00:55,035 AND YOU KNOW WHAT I FIND NOW, IN 6563 04:00:55,035 --> 04:00:57,337 THE LAST 10 YEARS, IS THAT 6564 04:00:57,337 --> 04:00:59,439 REFERRALS FOR PATIENTS WITH IPF 6565 04:00:59,439 --> 04:01:01,641 TO CLINICAL TRIALS, HAVE REALLY 6566 04:01:01,641 --> 04:01:02,409 FALLEN OFF. 6567 04:01:02,409 --> 04:01:04,377 THEY'RE DRIVING THEMSELVES TO 6568 04:01:04,377 --> 04:01:05,779 CLINICAL TRIAL ENROLLMENT AS 6569 04:01:05,779 --> 04:01:07,514 OPPOSE TO BEING RECOMMENDED BY 6570 04:01:07,514 --> 04:01:13,720 THEIR PRIMARY PULL 6571 04:01:13,720 --> 04:01:16,022 MONITORROLOGYIST, SO THERE ARE 6572 04:01:16,022 --> 04:01:17,290 LOTS OF IN-ROADS TO THE 6573 04:01:17,290 --> 04:01:17,891 HEALTHCARE PROFESSIONALS THAT 6574 04:01:17,891 --> 04:01:19,659 ARE IN THE COMMUNITY AND 6575 04:01:19,659 --> 04:01:22,929 EMPHASIZING CLINICAL TRIALS AS A 6576 04:01:22,929 --> 04:01:24,564 CARE OPTION AS PART OF THEIR 6577 04:01:24,564 --> 04:01:25,732 CLINICAL CARE FOR PATIENT WHO IS 6578 04:01:25,732 --> 04:01:27,501 ARE ELIGIBLE FOR THOSE CLINICAL 6579 04:01:27,501 --> 04:01:28,201 TRIALS. 6580 04:01:28,201 --> 04:01:29,302 IT'S SO IMPORTANT. 6581 04:01:29,302 --> 04:01:31,671 IT'S NOT AN EITHER OR, IT SHOULD 6582 04:01:31,671 --> 04:01:32,672 BE GOING ALONGSIDE. 6583 04:01:32,672 --> 04:01:34,741 YOU KNOW MANY IPF SITES AT THIS 6584 04:01:34,741 --> 04:01:36,776 POINT FOR AT LEAST SOME PORTION 6585 04:01:36,776 --> 04:01:38,311 OF PARTICIPANTS ALLOW BACKGROUND 6586 04:01:38,311 --> 04:01:40,580 THERAPY, MAKE IT A LITTLE MORE 6587 04:01:40,580 --> 04:01:43,750 CHALLENGING TO REACH THOSE END 6588 04:01:43,750 --> 04:01:46,152 POINTS, BUT, THAT SHOULD NOT 6589 04:01:46,152 --> 04:01:47,120 EXCLUDE PATIENT -- IT SHOULDN'T 6590 04:01:47,120 --> 04:01:48,822 BE THOUGHT OF AS I FAILED 6591 04:01:48,822 --> 04:01:50,357 EVERYTHING, NOW, WHAT'S THE 6592 04:01:50,357 --> 04:01:51,057 CLINICAL TRIAL OPTION. 6593 04:01:51,057 --> 04:01:52,893 I'VE PROGRESSED TO END OF LIFE, 6594 04:01:52,893 --> 04:01:53,593 NOW, WHAT'S MY CLINICAL TRIAL 6595 04:01:53,593 --> 04:01:55,562 OPTION, IT SHOULD BE AN EARLY 6596 04:01:55,562 --> 04:01:57,197 ON, YOU KNOW, THAT CAN GO 6597 04:01:57,197 --> 04:01:59,399 ALONGSIDE WITH ALL OF THE OTHER 6598 04:01:59,399 --> 04:02:00,133 APPROPRIATE LYNNICAL CARE THAT 6599 04:02:00,133 --> 04:02:01,167 PEOPLE ARE GETTING AND THEN I 6600 04:02:01,167 --> 04:02:04,404 WILL GO BACK TO WHAT TRE WAS 6601 04:02:04,404 --> 04:02:06,106 SAYING, I THINK SHARED DECISION 6602 04:02:06,106 --> 04:02:10,277 MAKING AS OPPOSE TO SORT OF A 6603 04:02:10,277 --> 04:02:11,678 MORE PATERNALISTIC WAY OF 6604 04:02:11,678 --> 04:02:12,412 LOOKING AT THINGS. 6605 04:02:12,412 --> 04:02:13,713 IT'S NOT -- IT'S NOT INFORMING 6606 04:02:13,713 --> 04:02:16,349 AND THEN SAYING THIS IS WHAT YOU 6607 04:02:16,349 --> 04:02:16,516 DO. 6608 04:02:16,516 --> 04:02:19,052 THIS IS A SHARED DECISION, THIS 6609 04:02:19,052 --> 04:02:20,487 IS PRESENTING OPTIONS AND 6610 04:02:20,487 --> 04:02:21,988 ASSESSING VALUES AND KNOWING 6611 04:02:21,988 --> 04:02:24,591 WHERE PEOPLE NEED TO BE AND 6612 04:02:24,591 --> 04:02:27,327 WHAT'S IMPORTANT FOR THEM AND I 6613 04:02:27,327 --> 04:02:29,629 THINK THAT APPROACH IS MUCH MORE 6614 04:02:29,629 --> 04:02:30,931 LIKELY TO YIELD SOMEBODY WHO HAS 6615 04:02:30,931 --> 04:02:32,766 NOR TRUST BOTH IN THE MEDICAL 6616 04:02:32,766 --> 04:02:41,441 SYSTEM AS WELL AS IN A CLINICAL 6617 04:02:41,441 --> 04:02:42,576 TRIAL OPPORTUNITY. 6618 04:02:42,576 --> 04:02:44,744 >> I WILL CONTINUE TO ENCOURAGE 6619 04:02:44,744 --> 04:02:46,947 PEOPLE TO POST QUESTIONS IN THE 6620 04:02:46,947 --> 04:02:48,648 Q&A TOOL AND MAYBE I WILL ASK 6621 04:02:48,648 --> 04:02:49,215 ANOTHER QUESTION. 6622 04:02:49,215 --> 04:02:50,350 YOU KNOW A LOT OF THINGS WE 6623 04:02:50,350 --> 04:02:54,588 TALKED ABOUT ARE THE LIMITATIONS 6624 04:02:54,588 --> 04:02:55,855 OF EFFECTIVE BIOMARKERS FOR SOME 6625 04:02:55,855 --> 04:02:58,091 OF THESE RARE DECS AND 6626 04:02:58,091 --> 04:03:01,528 CONVINCING THE FDA, WHAT IS A 6627 04:03:01,528 --> 04:03:03,029 RELEVANT BIOMARKER AND THAT CAN 6628 04:03:03,029 --> 04:03:04,898 BE A MYRIAD OF THINGS YOU 6629 04:03:04,898 --> 04:03:06,566 DISCUSSED, IT COULD BE BLOOD 6630 04:03:06,566 --> 04:03:07,968 BASED, IMAGE BASED BUT THEN ALSO 6631 04:03:07,968 --> 04:03:10,403 THINKING ABOUT PATIENT REPORTED 6632 04:03:10,403 --> 04:03:11,805 OUTCOMES AND SO, I WONDERED IF 6633 04:03:11,805 --> 04:03:13,573 YOU COULD TALK ABOUT A LITTLE 6634 04:03:13,573 --> 04:03:15,208 BIT OF THOSE MODALITIES FOR SOME 6635 04:03:15,208 --> 04:03:17,577 OF THE RARE DISEASES AND HOW 6636 04:03:17,577 --> 04:03:21,214 THEY MAY BE MOVING CLOSER TO 6637 04:03:21,214 --> 04:03:22,716 ACCEPTANCE BY NOT ONLY THE 6638 04:03:22,716 --> 04:03:25,785 RESEARCH COMMUNITY AS VALIDATED 6639 04:03:25,785 --> 04:03:27,420 BY BIOMARKERS BUT POTENTIALLY 6640 04:03:27,420 --> 04:03:29,155 FOR THE FDA AND IN PARTICULAR 6641 04:03:29,155 --> 04:03:31,891 HOW AN INDIVIDUAL MAY FEEL AND 6642 04:03:31,891 --> 04:03:33,693 THEY PERCEIVE THEIR HEALTH 6643 04:03:33,693 --> 04:03:35,562 STATUS AFTER AN INTERVENTION MAY 6644 04:03:35,562 --> 04:03:37,297 ALSO BE A REALLY GOOD MEANINGFUL 6645 04:03:37,297 --> 04:03:38,832 OUTCOME AND IT'S NOT SOMETHING 6646 04:03:38,832 --> 04:03:40,266 THAT WE TOUCHED A LOT DURING THE 6647 04:03:40,266 --> 04:03:41,835 SESSION, SO IF WE COULD TALK 6648 04:03:41,835 --> 04:03:50,310 ABOUT THAT A LITTLE BIT MORE, I 6649 04:03:50,310 --> 04:03:50,877 WOULD APPRECIATE IT. 6650 04:03:50,877 --> 04:03:52,746 >> I'LL GO AHEAD AND JUST SAY 6651 04:03:52,746 --> 04:03:55,949 THAT I THINK, YOU KNOW, I WAS 6652 04:03:55,949 --> 04:03:57,484 WATCHING WITH BAITED BREATH WHEN 6653 04:03:57,484 --> 04:04:00,820 THE FDA STARTED RELEASING THE 6654 04:04:00,820 --> 04:04:01,955 PATIENT FOCUSED DRUG DEVELOPMENT 6655 04:04:01,955 --> 04:04:05,759 GUIDANCE A LOT FOR THIS REASON A 6656 04:04:05,759 --> 04:04:09,529 LOT AND THE TYPICAL FEELS 6657 04:04:09,529 --> 04:04:11,264 FUNCTION AND SURVIVES PARADIGM, 6658 04:04:11,264 --> 04:04:13,433 IT'S HARD PARSING OUT HOW 1 6659 04:04:13,433 --> 04:04:14,701 FEEL'S IS ALSO, I DIDN'T TALK 6660 04:04:14,701 --> 04:04:16,569 ABOUT THIS DURING MY TALK BUT 6661 04:04:16,569 --> 04:04:20,173 I'VE EXPERIENCED AND LIVED WITH 6662 04:04:20,173 --> 04:04:21,107 ANXIETY AND DEPRESSION FOR QUITE 6663 04:04:21,107 --> 04:04:22,409 SOMETIME AND YOU CAN'T REALLY 6664 04:04:22,409 --> 04:04:24,144 SEPARATE WHEN YOU'RE DEALING 6665 04:04:24,144 --> 04:04:25,311 WITH CHRONIC CONDITIONS YOU 6666 04:04:25,311 --> 04:04:26,846 CANNOT SEPARATE ALL THESE 6667 04:04:26,846 --> 04:04:27,614 EXTERNALITIES FROM HOW SOMEBODY 6668 04:04:27,614 --> 04:04:30,817 FEELS AND I THINK IT'S REALLY 6669 04:04:30,817 --> 04:04:32,485 IMPORTANT THAT THE FIELD 6670 04:04:32,485 --> 04:04:33,319 PRIORITIZES VALIDATION AND 6671 04:04:33,319 --> 04:04:36,022 UNDERSTANDING OF PATIENT 6672 04:04:36,022 --> 04:04:37,991 REPORTED OUTCOMES AND I THINK OF 6673 04:04:37,991 --> 04:04:40,727 THE FDA A LOT AS A HELP ME HELP 6674 04:04:40,727 --> 04:04:42,295 YOU SITUATION WHERE I THINK THE 6675 04:04:42,295 --> 04:04:44,497 FDA IS EAGER TO MOVE THINGS IN 6676 04:04:44,497 --> 04:04:47,000 AN INNOVATIVE DIRECTION BUT I 6677 04:04:47,000 --> 04:04:47,734 THINK IT'S REALLY INCUMBENT ON 6678 04:04:47,734 --> 04:04:49,035 THE FIELD TO COME TOGETHER TO 6679 04:04:49,035 --> 04:04:51,337 SORT OF DRIVE AND GENERATE THAT 6680 04:04:51,337 --> 04:04:53,306 DATA TO DEMONSTRATE AND YOU KNOW 6681 04:04:53,306 --> 04:04:55,041 THERE'S A WHOLE FIELD OF 6682 04:04:55,041 --> 04:04:56,443 INVESTIGATORRING THE PLACEBO 6683 04:04:56,443 --> 04:04:57,510 EFFECT AND UNDERSTANDING, 6684 04:04:57,510 --> 04:04:58,778 PARSING OUT WHAT'S HAPPENING 6685 04:04:58,778 --> 04:05:00,747 WHEN PEOPLE REPORT IMPROVEMENTS 6686 04:05:00,747 --> 04:05:05,952 WITH THE PLACEBO, WITH PLACEBO, 6687 04:05:05,952 --> 04:05:07,253 BUT ULTIMATELY, IT'S -- I OFTEN 6688 04:05:07,253 --> 04:05:08,888 -- WHEN I TALK TO PATIENTS ABOUT 6689 04:05:08,888 --> 04:05:10,223 THIS, I TALK ABOUT PATIENT 6690 04:05:10,223 --> 04:05:13,226 REPORTED OUT COMES AS WE DON'T 6691 04:05:13,226 --> 04:05:14,728 WANT -- IT'S NOT GOOD FOR 6692 04:05:14,728 --> 04:05:15,462 PATIENT COMMUNITIES IN MY VIEW 6693 04:05:15,462 --> 04:05:17,997 TO COME TO A ARE AND SAY THIS 6694 04:05:17,997 --> 04:05:19,065 TREATMENT'S NOT WORKING AND THEN 6695 04:05:19,065 --> 04:05:20,533 EXPECT THAT RELATIONSHIP TO JUST 6696 04:05:20,533 --> 04:05:22,669 BE THAT THE DOCTOR SAYS, OKAY 6697 04:05:22,669 --> 04:05:23,603 THAT'S DRUG'S NOT WORKING NOT 6698 04:05:23,603 --> 04:05:24,738 SAYING THAT THE PATIENT SHOULD 6699 04:05:24,738 --> 04:05:25,939 NOT HAVE THEIR EXPERIENCE 6700 04:05:25,939 --> 04:05:27,941 VALIDATED BUT I THINK THE POWER 6701 04:05:27,941 --> 04:05:32,512 OF PATIENT REPORTED OUTCOMES IS 6702 04:05:32,512 --> 04:05:33,680 A METHOD LOGICAL APPROACH TO,A 6703 04:05:33,680 --> 04:05:35,648 ASSESSES HOW PEOPLE ARE FEELING 6704 04:05:35,648 --> 04:05:38,351 IN A WAY THAT GETS INTO MAYBE 6705 04:05:38,351 --> 04:05:44,124 THE MORE INEFFABLE PARTS OF 6706 04:05:44,124 --> 04:05:45,558 MEDICATION, I DON'T KNOW IF THAT 6707 04:05:45,558 --> 04:05:47,460 ANSWERS YOUR QUESTION MARRAH, 6708 04:05:47,460 --> 04:05:48,561 BUT THAT'S MY PERSPECTIVE ON 6709 04:05:48,561 --> 04:05:49,963 PATIENT REPORTED OUT COMES. 6710 04:05:49,963 --> 04:05:51,097 THEY'RE LIKE ANYTHING ELSE A 6711 04:05:51,097 --> 04:05:52,732 TOOL THAT CAN BE POWERFUL TO 6712 04:05:52,732 --> 04:05:54,234 STRENGTHEN THE CASE OF 6713 04:05:54,234 --> 04:05:55,535 THERAPEUTICS. 6714 04:05:55,535 --> 04:05:59,038 >> YEAH AND I THINK 1 CHALLENGE 6715 04:05:59,038 --> 04:06:00,907 WE HAVE TO UNDERSTANDING EARLIER 6716 04:06:00,907 --> 04:06:02,208 DISEASE AND DEVELOPING 6717 04:06:02,208 --> 04:06:03,743 BIOMARKERS IS THAT IF WE JUST 6718 04:06:03,743 --> 04:06:04,778 START FOLLOWING ANYBODY WHO HAS 6719 04:06:04,778 --> 04:06:07,313 SOMETHING ON A SCAN, YOU KNOW 10 6720 04:06:07,313 --> 04:06:09,015 OR 20% MIGHT GO ON SO HAVE YOU A 6721 04:06:09,015 --> 04:06:09,549 LOT OF NOISE. 6722 04:06:09,549 --> 04:06:12,318 SO CAN WE FIGURE OUT WHO THOSE 6723 04:06:12,318 --> 04:06:12,619 PEOPLE ARE? 6724 04:06:12,619 --> 04:06:14,187 SO AGAIN, WE'RE LOOKING BACK, 6725 04:06:14,187 --> 04:06:16,956 AND WE'RE TRYING TO ANALYZE THE 6726 04:06:16,956 --> 04:06:19,459 HRCT SCANS FROM WHAT I CALL 6727 04:06:19,459 --> 04:06:20,527 PRESEIZE, WE DON'T HAVE GOOD 6728 04:06:20,527 --> 04:06:23,997 LANGUAGE TO DESCRIBE THIS, CAN 6729 04:06:23,997 --> 04:06:26,232 WE SEE SOMETHING THERE WITH A 6730 04:06:26,232 --> 04:06:26,866 NEW TECHNOLOGY, RIGHT? 6731 04:06:26,866 --> 04:06:32,038 SO THEY'VE BEEN LOOKED AT 10 6732 04:06:32,038 --> 04:06:34,174 YEARS AGO 12 YEARS AGO COULD WE 6733 04:06:34,174 --> 04:06:36,376 SEE ANYTHING THAT WE COULD THEN 6734 04:06:36,376 --> 04:06:38,444 ENRICH A POPULATION TO FOLLOW OR 6735 04:06:38,444 --> 04:06:39,879 HOPEFULLY FIND OR FOLLOW A 6736 04:06:39,879 --> 04:06:41,414 BIOMARKER SO YOU COULD INCREASE 6737 04:06:41,414 --> 04:06:42,215 YOUR RISK SCORE. 6738 04:06:42,215 --> 04:06:46,286 SO THERE'S A LOT OF PLACES WE'VE 6739 04:06:46,286 --> 04:06:49,155 DONE WORK WITH THE LAHEY CLINIC 6740 04:06:49,155 --> 04:06:51,591 WHERE IF YOU HAVE AN ILA YOU GO 6741 04:06:51,591 --> 04:06:53,459 TO A LUNG HEALTH CLINIC BUT 6742 04:06:53,459 --> 04:06:54,994 AGAIN THAT COULD SCARE PEOPLE, 6743 04:06:54,994 --> 04:06:56,396 YOU HAVE AN ILA YOU HAVE TO GO 6744 04:06:56,396 --> 04:06:58,464 TO THE LUNG CLINIC SO WHO SHOULD 6745 04:06:58,464 --> 04:06:59,599 GO AND WHO SHOULDN'T? 6746 04:06:59,599 --> 04:07:02,669 SO CAN WE GET A BIOMARKER RISK 6747 04:07:02,669 --> 04:07:04,070 SCORE, WHERE WE CAN PREYECT MORE 6748 04:07:04,070 --> 04:07:06,239 WHO WILL PROGRESS AND GET THEM 6749 04:07:06,239 --> 04:07:07,273 IN EARLIER AND IT'S ONLY GOING 6750 04:07:07,273 --> 04:07:11,010 TO BE DONE IF WE CAN FOLLOW OVER 6751 04:07:11,010 --> 04:07:13,146 TIME AND TOO, IF WE CAN GET THEM 6752 04:07:13,146 --> 04:07:14,314 EARLIER, THAT WOULD BE GREAT BUT 6753 04:07:14,314 --> 04:07:15,815 THEN HOW DO WE DO A CLINICAL 6754 04:07:15,815 --> 04:07:16,683 TRIAL. 6755 04:07:16,683 --> 04:07:17,884 WE NEED VALIDATED BIOMARKERS SO 6756 04:07:17,884 --> 04:07:19,185 SOMEHOW WE HAVE TO START DOING 6757 04:07:19,185 --> 04:07:21,588 THAT NOW BECAUSE IT'S SUCH A 6758 04:07:21,588 --> 04:07:24,424 LONG PROCESS, SO WHAT CAN 6759 04:07:24,424 --> 04:07:26,125 INDUSTRY BE PUTTING INTO THEIR 6760 04:07:26,125 --> 04:07:29,762 CLINICAL TRIALS OR LOOKING AT IN 6761 04:07:29,762 --> 04:07:31,531 AITITION TO FVC IN EAPGZ FOR THE 6762 04:07:31,531 --> 04:07:31,764 FUTURE. 6763 04:07:31,764 --> 04:07:39,272 I MEAN WE HAVE TO START NOW. 6764 04:07:39,272 --> 04:07:40,874 SMRKS SO WE'VE HAD A LITTLE 6765 04:07:40,874 --> 04:07:42,542 TRIEWBL AND THIS GOES TO 6766 04:07:42,542 --> 04:07:43,176 PREDISEASE STATE. 6767 04:07:43,176 --> 04:07:45,478 FOR SOME OF THESE DECS THERE'S 6768 04:07:45,478 --> 04:07:46,913 NO PREDEC STATE TO PROVIDE A 6769 04:07:46,913 --> 04:07:47,580 BASE LINE. 6770 04:07:47,580 --> 04:07:50,283 SO WE HAVE A VALIDATED TOOL, A 6771 04:07:50,283 --> 04:07:52,252 HEALTHCARA QUALITY OF LIFE FOR 6772 04:07:52,252 --> 04:07:53,953 PATIENTS BUT IT'S NOT BEEN 6773 04:07:53,953 --> 04:07:55,588 EFFECTIVE BUT WHEN YOU'RE 6774 04:07:55,588 --> 04:07:57,657 SYMPTOMATIC EVERY SINGLE DAY AND 6775 04:07:57,657 --> 04:07:58,925 YOU'RE ASKING PEOPLE HOW THEY 6776 04:07:58,925 --> 04:08:01,160 FEEL FOR A 1 OR 2 WEEK PERIOD, 6777 04:08:01,160 --> 04:08:02,795 IT HASN'T BEEN PARTICULARLY 6778 04:08:02,795 --> 04:08:03,129 ENLIGHTENING. 6779 04:08:03,129 --> 04:08:05,565 THE ISSUE WE HAVE IS FINDING 6780 04:08:05,565 --> 04:08:07,834 GOOD VALIDATED PATIENT REPORTED 6781 04:08:07,834 --> 04:08:08,101 OUTCOMES. 6782 04:08:08,101 --> 04:08:09,068 IT'S NOT THAT WE'RE NOT 6783 04:08:09,068 --> 04:08:10,803 INTERESTED IN DOING IT, WE 6784 04:08:10,803 --> 04:08:15,642 HAVEN'T FOUND THE RIGHT 6785 04:08:15,642 --> 04:08:15,975 MECHANISM. 6786 04:08:15,975 --> 04:08:18,278 >> AND I'LL JUST SAY A FEW WORDS 6787 04:08:18,278 --> 04:08:21,047 ABOUT THIS, VEG FD IS A POWERFUL 6788 04:08:21,047 --> 04:08:21,748 BIOMARKER. 6789 04:08:21,748 --> 04:08:23,249 IT'S A POWERFUL BIOMARKER. 6790 04:08:23,249 --> 04:08:26,786 IF IT'S POSITIVE THE SPECIFICITY 6791 04:08:26,786 --> 04:08:27,887 IS 98%. 6792 04:08:27,887 --> 04:08:30,323 EVEN IF IT'S WITHIN, YOU KNOW 6793 04:08:30,323 --> 04:08:32,158 20% OF POSITIVE, IT'S STILL 6794 04:08:32,158 --> 04:08:33,660 BETTER THAN 90% SPECIFIC. 6795 04:08:33,660 --> 04:08:35,061 IT'S NOT VERY SENSITIVE, THIS IS 6796 04:08:35,061 --> 04:08:38,965 ABOUT HALF OF PATIENTS. 6797 04:08:38,965 --> 04:08:41,801 AS A PROGNOSTIC BIOMARKER, IT'S 6798 04:08:41,801 --> 04:08:43,603 GOT SOME VALUE, BUT NO VALUE AS 6799 04:08:43,603 --> 04:08:45,672 AN END POINT AT ALL, SO THERE 6800 04:08:45,672 --> 04:08:47,073 ISN'T -- DESPITE A LOT OF 6801 04:08:47,073 --> 04:08:48,708 RESEARCH, THERE ISN'T A 6802 04:08:48,708 --> 04:08:50,109 BIOMARKER I COULD APPROACH THE 6803 04:08:50,109 --> 04:08:52,312 FDA ABOUT THAT WOULD BE A 6804 04:08:52,312 --> 04:08:53,479 SUITABLE END POINT AND I WOULD 6805 04:08:53,479 --> 04:08:55,581 SUGGEST THAT'S TRUE FOR IPP AS 6806 04:08:55,581 --> 04:08:57,116 WELL, IT ISN'T AS IF WE'RE GOING 6807 04:08:57,116 --> 04:08:58,985 TO THE FDA WITH PLAUSIBLE 6808 04:08:58,985 --> 04:09:01,254 CANDIDATES AND BEING REJECTED 6809 04:09:01,254 --> 04:09:03,556 FOR, YOU KNOW UNJUST REASONS, 6810 04:09:03,556 --> 04:09:04,791 IT'S THAT WE HAVEN'T CONVINCED 6811 04:09:04,791 --> 04:09:07,193 OURSELVES THAT WE ARE A 6812 04:09:07,193 --> 04:09:09,295 BIOMARKER THAT IS A SUITABLE END 6813 04:09:09,295 --> 04:09:09,495 POINT. 6814 04:09:09,495 --> 04:09:14,100 THAT'S MY PERSPECTIVE ON THINGS. 6815 04:09:14,100 --> 04:09:15,635 SO, I THINK TRE'S EXACTLY RIGHT, 6816 04:09:15,635 --> 04:09:17,971 THEY WANT TO BE HELPFUL, WE HAVE 6817 04:09:17,971 --> 04:09:21,507 TO BRING THEM SOMETHING THAT'S 6818 04:09:21,507 --> 04:09:21,975 ACTUALLY APPROVABLE. 6819 04:09:21,975 --> 04:09:24,077 >> I THINK SOME OF THE 6820 04:09:24,077 --> 04:09:27,447 CANDIDATES COULD BE FIGURING OUT 6821 04:09:27,447 --> 04:09:29,716 CT SCANS, RIGHT BECAUSE THEY ARE 6822 04:09:29,716 --> 04:09:33,019 TIED TO DISEASE BUT YET THEY'RE 6823 04:09:33,019 --> 04:09:35,755 NOT PREDICTIVE OR IT HASPT BEEN 6824 04:09:35,755 --> 04:09:38,358 VALIDATED AND I THINK THOSE ARE 6825 04:09:38,358 --> 04:09:39,759 BEING DONE IN TRIALS ALL THE 6826 04:09:39,759 --> 04:09:41,728 TIME SO FIGURE THAT OUT AND THEN 6827 04:09:41,728 --> 04:09:45,164 THERE MIGHT BE SOME COLLEGE AND 6828 04:09:45,164 --> 04:09:46,666 BI-PRODUCTS THAT BE LOOKED AT. 6829 04:09:46,666 --> 04:09:48,201 SO THERE ARE SOME THINGS THAT 6830 04:09:48,201 --> 04:09:49,502 COULD BE LOOKED AT AND I KNOW 6831 04:09:49,502 --> 04:09:51,004 THERE WAS A DISCUSSION A YEAR 6832 04:09:51,004 --> 04:09:53,106 AND HALF AGO WITH THE FDA AND 6833 04:09:53,106 --> 04:09:54,207 THEY TALKED MORE ABOUT FOCUSING 6834 04:09:54,207 --> 04:09:55,875 ON THE PATIENT REPORTED OUT 6835 04:09:55,875 --> 04:09:57,343 COMES, I BELIEVE, I DON'T KNOW, 6836 04:09:57,343 --> 04:10:00,113 AMY YOU MAY KNOW MORE ABOUT THAT 6837 04:10:00,113 --> 04:10:01,781 THAN I DO BUT I THINK WE HAVE TO 6838 04:10:01,781 --> 04:10:07,487 KEEP THINKING ABOUT THIS. 6839 04:10:07,487 --> 04:10:09,389 >> YEAH, I THINK THAT I 6840 04:10:09,389 --> 04:10:10,056 COMPLETELY AGREE. 6841 04:10:10,056 --> 04:10:14,794 I THINK 1 OF THE PROBLEMS WITH 6842 04:10:14,794 --> 04:10:16,529 THE PATIENT REPORTED OUT COME AS 6843 04:10:16,529 --> 04:10:18,698 A COMMUNITY HAVEN'T GENERATED 6844 04:10:18,698 --> 04:10:21,134 ENOUGH DATA TO VALIDATE IT IT'S 6845 04:10:21,134 --> 04:10:23,736 LIKE PROs ARE LIKE ICE CREAM, 6846 04:10:23,736 --> 04:10:25,004 EVERYBODY'S GOT THEIR OWN 6847 04:10:25,004 --> 04:10:25,471 FAVORITE FLAVOR. 6848 04:10:25,471 --> 04:10:28,241 SO ACROSS STUDIES THEY'RE NOT 6849 04:10:28,241 --> 04:10:32,278 UTILIZING AND BUILDING THE DATA 6850 04:10:32,278 --> 04:10:34,514 TOWARDS, YOU KNOW ANYONE PATIENT 6851 04:10:34,514 --> 04:10:35,148 REPORTED OUTCOME. 6852 04:10:35,148 --> 04:10:39,986 SO I THINK THAT IT IS INCUMBENT 6853 04:10:39,986 --> 04:10:41,354 UPON THE COMMUNITY IN BRINGS 6854 04:10:41,354 --> 04:10:42,688 THESE DIFFERENT VOICES TOGETHER. 6855 04:10:42,688 --> 04:10:47,493 I THINK GETTING BACK TO PATIENT 6856 04:10:47,493 --> 04:10:48,094 ADVOCACY ORGANIZATIONS WE'RE 6857 04:10:48,094 --> 04:10:50,763 PRIMED TO DO THAT, EACH OF OUR 6858 04:10:50,763 --> 04:10:51,431 ORGANIZATION SYSTEM DEMONSTRATED 6859 04:10:51,431 --> 04:10:55,101 AN ABILITY TO BRING TOGETHER 6860 04:10:55,101 --> 04:10:57,370 DIFFERENT VIEW POINTS, DIFFERENT 6861 04:10:57,370 --> 04:10:58,771 ORGANIZATIONS TO WORK TOGETHER 6862 04:10:58,771 --> 04:11:00,473 TO THEY NOT NATURALLY OTHERWISE 6863 04:11:00,473 --> 04:11:02,642 DO SO AND I THINK THERE'S A LOT 6864 04:11:02,642 --> 04:11:09,582 OF INTEREST IN VALIDATING PROs 6865 04:11:09,582 --> 04:11:11,551 FOR PULMONARY FIBROSIS BECAUSE, 6866 04:11:11,551 --> 04:11:16,055 YOU KNOW FVC IS NOT NECESSARILY 6867 04:11:16,055 --> 04:11:18,257 A FEELS OR FUNCTIONS END POINT 6868 04:11:18,257 --> 04:11:19,892 FOR MANY PATIENTS SO IT DOESN'T 6869 04:11:19,892 --> 04:11:23,062 NECESSARILY TELL THAT TRUTH FOR 6870 04:11:23,062 --> 04:11:23,296 PEOPLE. 6871 04:11:23,296 --> 04:11:24,530 >> IF IT'S OKAY, I WOULD LIKE TO 6872 04:11:24,530 --> 04:11:27,800 ASK A QUESTION OF THE PANEL. 6873 04:11:27,800 --> 04:11:32,171 EACH OF THE DIFFERENT 6874 04:11:32,171 --> 04:11:33,406 ORGANIZATIONS HERE HAS REGISTRY 6875 04:11:33,406 --> 04:11:36,476 OR YOU KNOW PATIENT REGISTRY 6876 04:11:36,476 --> 04:11:36,976 EFFORTS. 6877 04:11:36,976 --> 04:11:40,012 I THINK THE CF FOUNDATION HAS 6878 04:11:40,012 --> 04:11:42,515 THE MOST ROBUST AND MOST 6879 04:11:42,515 --> 04:11:43,783 EXPERIENCED IN THIS AND THERE'S 6880 04:11:43,783 --> 04:11:45,551 A LOT TO BE LEARN FRIDAY THAT 6881 04:11:45,551 --> 04:11:47,186 BUT I ACTUALLY THINK THIS IS 1 6882 04:11:47,186 --> 04:11:49,355 OF THE MOST IMPORTANT THINGS AS 6883 04:11:49,355 --> 04:11:52,291 WE'RE MOVING FORWARD IS FOR 6884 04:11:52,291 --> 04:11:54,961 THERE TO BE EITHER DECENTRALIZED 6885 04:11:54,961 --> 04:11:56,429 OR MULTICENTER LARGE DATA SETS 6886 04:11:56,429 --> 04:11:59,198 BECAUSE WE ARE IN AN ERA OF 6887 04:11:59,198 --> 04:12:01,134 LARGE DATA AND TRYING TO 6888 04:12:01,134 --> 04:12:02,068 UNDERSTAND THESE THINGS AND 6889 04:12:02,068 --> 04:12:04,070 THEN, YOU KNOW WE COME UP WITH 6890 04:12:04,070 --> 04:12:06,139 THESE NEW IDEAS, THEY NEED TO BE 6891 04:12:06,139 --> 04:12:08,908 -- THEY NEED TO BE INVESTIGATED 6892 04:12:08,908 --> 04:12:10,042 FURTHER, INTERROGATED FURTHER, 6893 04:12:10,042 --> 04:12:11,744 VALIDATED FURTHER AND SO TO HAVE 6894 04:12:11,744 --> 04:12:13,513 SOMETHING THAT'S VERY WELL 6895 04:12:13,513 --> 04:12:14,714 CLINICALLY CHARACTERIZED AND 6896 04:12:14,714 --> 04:12:19,886 THEN ALSO HAS THOSE BIOLOGICAL 6897 04:12:19,886 --> 04:12:21,420 INFORMATION ASSOCIATE WIDE IT, I 6898 04:12:21,420 --> 04:12:23,055 THINK IS REALLY IMPORTANT. 6899 04:12:23,055 --> 04:12:26,092 SO MAYBE TALK A LITTLE BIT ABOUT 6900 04:12:26,092 --> 04:12:26,425 THAT. 6901 04:12:26,425 --> 04:12:29,295 WE HAVE SMALL, YOU KNOW, SMALL 6902 04:12:29,295 --> 04:12:30,596 GROUPS OF OUR RESPECTIVE 6903 04:12:30,596 --> 04:12:32,098 PATIENTS SO GETTING THOSE DATA 6904 04:12:32,098 --> 04:12:34,167 TOGETHER I THINK IS JUST VITALLY 6905 04:12:34,167 --> 04:12:34,433 IMPORTANT. 6906 04:12:34,433 --> 04:12:37,737 SO I WOULD LIKE TO HEAR FROM THE 6907 04:12:37,737 --> 04:12:38,905 EXPERIENCE THERE FROM THE OTHER 6908 04:12:38,905 --> 04:12:41,440 GROUPS IF I COULD. 6909 04:12:41,440 --> 04:12:44,510 >> IT TOOK US A LONG TIME TO 6910 04:12:44,510 --> 04:12:45,578 LAUNCH A REGISTRY BECAUSE WE 6911 04:12:45,578 --> 04:12:47,079 KNEW WE WANTED A CLINICAL REG 6912 04:12:47,079 --> 04:12:48,648 TREE AND WE KNEW WE WANTED IT TO 6913 04:12:48,648 --> 04:12:49,916 BE COLLECTED THROUGH THE SITES 6914 04:12:49,916 --> 04:12:52,485 BUT WE ALSO KNOW, THIS IS A 6915 04:12:52,485 --> 04:12:53,519 SMALL SUBSET OF OUR PATIENT 6916 04:12:53,519 --> 04:12:58,157 GROUP, SO WE ARE LOOKING NOW, TO 6917 04:12:58,157 --> 04:13:00,459 DO A BIG BROAD FUNNEL AND BRING 6918 04:13:00,459 --> 04:13:03,763 PEOPLE IN, MAYBE TO PATIENT 6919 04:13:03,763 --> 04:13:05,364 REPORTED REGISTRIES BUT I THINK 6920 04:13:05,364 --> 04:13:07,433 THE 1 THING THAT IT KIND OF 6921 04:13:07,433 --> 04:13:08,834 GETTING BACK TO RESOURCES AND 6922 04:13:08,834 --> 04:13:10,236 HOW MANY OF THESE RARE DEC 6923 04:13:10,236 --> 04:13:14,173 GROUPS ARE SO SMALL, REGISTRIES 6924 04:13:14,173 --> 04:13:15,942 ARE INCREDIBLY EBS PENSIVE AND I 6925 04:13:15,942 --> 04:13:17,210 THINK WITHOUT SUPPORT, IT'S JUST 6926 04:13:17,210 --> 04:13:18,511 SHOCKING TO ME HOW CRITICALLY 6927 04:13:18,511 --> 04:13:19,912 IMPORTANT THEY ARE AND YET IT IS 6928 04:13:19,912 --> 04:13:22,648 REALLY HARD TO GET THEM FUNDED. 6929 04:13:22,648 --> 04:13:24,217 AND I THINK WE'RE MISSING, I 6930 04:13:24,217 --> 04:13:25,518 THINK WE'RE MISSING A LOT OF 6931 04:13:25,518 --> 04:13:28,187 REALLY IMPORTANT DATA FROM A LOT 6932 04:13:28,187 --> 04:13:34,427 OF DIFFERENT DISEASES BECAUSE F 6933 04:13:34,427 --> 04:13:34,627 THAT. 6934 04:13:34,627 --> 04:13:36,629 >> I WILL SECOND HOW EFFORT 6935 04:13:36,629 --> 04:13:38,965 INTENSIVE MAINTENANCE OF THESE 6936 04:13:38,965 --> 04:13:40,066 REGISTRIES IS, OUR CLINICAL 6937 04:13:40,066 --> 04:13:41,167 TRIALS COORDINATORS DO IT MORE 6938 04:13:41,167 --> 04:13:44,637 OR LESS OUT OF THE GOODNESS OF 6939 04:13:44,637 --> 04:13:46,806 THEIR HEARTS BECAUSE IT'S REALLY 6940 04:13:46,806 --> 04:13:47,640 UNDERFUNDED AND IT'S HARD TO 6941 04:13:47,640 --> 04:13:50,042 KEEP ALL THE HOLES FILLED AND I 6942 04:13:50,042 --> 04:13:51,777 THINK FOR RARE DISEASES AND SOME 6943 04:13:51,777 --> 04:13:53,679 WAYS IT MIGHT MAKE MORE SENSE TO 6944 04:13:53,679 --> 04:13:55,481 HAVE A CONTACT REGISTRY WITHOUT 6945 04:13:55,481 --> 04:13:58,918 A LOT OF DEPTH AND IDENTIFY 6946 04:13:58,918 --> 04:14:00,853 WHERE PEOPLE ARE AND A LITTLE 6947 04:14:00,853 --> 04:14:02,321 BIT OF PHENOTYPIC INFORMATION 6948 04:14:02,321 --> 04:14:03,422 ABOUT THEM AND THEN WHEN IT 6949 04:14:03,422 --> 04:14:04,790 COMES TO TIME FOR THEM TO DO A 6950 04:14:04,790 --> 04:14:06,058 STUDY, YOU KNOW WHERE THEY ARE, 6951 04:14:06,058 --> 04:14:08,928 YOU CAN CONTACT THEM, CAN YOU 6952 04:14:08,928 --> 04:14:09,662 ANSWER SPECIFIC QUESTIONS, BUT 6953 04:14:09,662 --> 04:14:11,530 THE PROCESS OF COLLECTING 6954 04:14:11,530 --> 04:14:13,599 DETAILED DATA WITHOUT A QUESTION 6955 04:14:13,599 --> 04:14:16,535 IN MIND IS SOMETIMES KIND OF A 6956 04:14:16,535 --> 04:14:17,737 FOOL'S ERRAND AND YOU END UP 6957 04:14:17,737 --> 04:14:23,776 WITH A LARGE AMOUNT OF DATA THAT 6958 04:14:23,776 --> 04:14:24,710 NOBODY USES. 6959 04:14:24,710 --> 04:14:26,646 >> THAT'S EXACTLY WHERE WE 6960 04:14:26,646 --> 04:14:26,879 LANDED. 6961 04:14:26,879 --> 04:14:28,414 WE'RE THINKING OF DOING THE BIG 6962 04:14:28,414 --> 04:14:30,283 FUNNEL APPROACH AS A CONTACT 6963 04:14:30,283 --> 04:14:31,917 DATABASE AND THEN WHEN THERE'S 6964 04:14:31,917 --> 04:14:32,952 SPECIFIC NEEDS, WE HAVE A 6965 04:14:32,952 --> 04:14:35,354 PATIENT GROUP WE CAN GO BACK TO 6966 04:14:35,354 --> 04:14:37,323 AND GET THE INFORMATION THAT WE 6967 04:14:37,323 --> 04:14:38,391 NEED, BUT, YEAH, IT'S A LOT OF 6968 04:14:38,391 --> 04:14:40,726 WORK AND A LOT OF MONEY FOR 6969 04:14:40,726 --> 04:14:44,263 PATIENT ENTRY INTO THESE THINGS. 6970 04:14:44,263 --> 04:14:45,331 >> THANK YOU ALL. 6971 04:14:45,331 --> 04:14:46,999 THAT CONCLUDES THE PANEL FOR 6972 04:14:46,999 --> 04:14:47,833 SESSION 5. 6973 04:14:47,833 --> 04:14:49,935 WE'RE MAKING SOME MODIFICATIONS 6974 04:14:49,935 --> 04:14:51,504 TO THE AGENDA TODAY, SO THAT WE 6975 04:14:51,504 --> 04:14:53,839 CAN END A LITTLE EARLIER. 6976 04:14:53,839 --> 04:14:55,841 INSTEAD OF HAVING A 30 MINUTE 6977 04:14:55,841 --> 04:14:57,143 NETWORKING BREAK, WE WILL TAKE A 6978 04:14:57,143 --> 04:14:59,145 15 MINUTE BREAK CAN THAT CAN BE 6979 04:14:59,145 --> 04:15:00,212 A COFFEE OR BIOBREAK IF YOU WISH 6980 04:15:00,212 --> 04:15:01,847 OR YOU CAN STAY IN THE ROOM. 6981 04:15:01,847 --> 04:15:05,751 AND WE ARE GOING TO RETURN AT 6982 04:15:05,751 --> 04:15:07,153 2:30 EASTERN TIME TO START THE 6983 04:15:07,153 --> 04:15:09,055 BREAK OUTS, SO I WILL DISCUSS 6984 04:15:09,055 --> 04:15:13,292 THOSE A BIT BEFORE YOU ALL CAN 6985 04:15:13,292 --> 04:15:14,694 SELECT WHICH BREAK OUT YOU WOULD 6986 04:15:14,694 --> 04:15:15,795 LIKE TO PARTICIPATE IN AND HOPE 6987 04:15:15,795 --> 04:15:17,730 YOU STAY ON AND YOIN 1 OF THOSE 6988 04:15:17,730 --> 04:15:18,964 GROUPS TO GIVE YOUR FEEDBACK 6989 04:15:18,964 --> 04:15:20,599 ABOUT THE NEXT STEPS FOR 6990 04:15:20,599 --> 04:15:21,667 ADVANCING THERAPIES INTO THE 6991 04:15:21,667 --> 04:15:25,256 CLINIC. THANK YOU ALL. 6992 04:15:25,256 --> 04:15:26,290 WE'RE GOING TO BE MOVING INTO 6993 04:15:26,290 --> 04:15:27,925 THE BREAKOUT SESSIONS BUT I JUST 6994 04:15:27,925 --> 04:15:29,260 WANTED TO SPEND A COUPLE OF 6995 04:15:29,260 --> 04:15:31,428 MINUTES GOING OVER EACH OF THEM. 6996 04:15:31,428 --> 04:15:33,063 IF WE COULD PULL UP THOSE 6997 04:15:33,063 --> 04:15:43,240 SLIDES.ES 6998 04:15:46,343 --> 04:15:48,045 SO WE HAVE THREE BREAKOUT 6999 04:15:48,045 --> 04:15:48,913 SESSIONS AND REALLY WHAT THESE 7000 04:15:48,913 --> 04:15:50,648 ARE DESIGNED TO DO IS TO GET 7001 04:15:50,648 --> 04:15:53,284 FEEDBACK FROM YOU ALL ON SOME OF 7002 04:15:53,284 --> 04:15:54,585 THE CENTRAL THEMES FROM THE 7003 04:15:54,585 --> 04:15:57,788 WORKSHOP OVER THE PAST TWO DAYS. 7004 04:15:57,788 --> 04:15:59,189 AND TO REALLY THINK ABOUT 7005 04:15:59,189 --> 04:16:00,624 CRITICAL BARRIERS AND 7006 04:16:00,624 --> 04:16:02,059 FACILITATORS TO ADVANCING 7007 04:16:02,059 --> 04:16:03,994 INNOVATIVE THERAPIES FROM IDEA 7008 04:16:03,994 --> 04:16:05,763 THROUGH COMMERCIALIZATION AND 7009 04:16:05,763 --> 04:16:07,264 THE IND PATHWAY. 7010 04:16:07,264 --> 04:16:09,466 AND SO WE HAVE THREE BREAKOUT 7011 04:16:09,466 --> 04:16:11,869 SESSIONS, BREAKOUT SESSION ONE 7012 04:16:11,869 --> 04:16:15,039 IS ENTITLED MODELS AND METHODS, 7013 04:16:15,039 --> 04:16:20,077 COMPUTATIONAL, INVIE T IN VITRON 7014 04:16:20,077 --> 04:16:22,046 VIVO, COVERING TOPICS FROM 7015 04:16:22,046 --> 04:16:22,980 TUESDAY'S SESSION ONE, OVERVIEW 7016 04:16:22,980 --> 04:16:25,416 OF THE DRUG AND DEVICEICDEVELOPE 7017 04:16:25,416 --> 04:16:26,817 DISCUSSED THE MODELS AND TOOLS 7018 04:16:26,817 --> 04:16:29,787 THAT THE PULMONARY AND SLEEP 7019 04:16:29,787 --> 04:16:30,654 MEDICINE RESEARCH COMMUNITY 7020 04:16:30,654 --> 04:16:32,222 COULD USE TO START THINKING 7021 04:16:32,222 --> 04:16:32,690 ABOUT 7022 04:16:32,690 --> 04:16:35,292 SCREENING FOR DRUG 7023 04:16:35,292 --> 04:16:36,493 CANDIDATES AND GETTING INTO SOME 7024 04:16:36,493 --> 04:16:39,630 OF THOSE PRE-CLINICAL 7025 04:16:39,630 --> 04:16:44,268 IND-ENABLING STUDIES. 7026 04:16:44,268 --> 04:16:46,337 THE NEXT BREAKOUT SESSION IS 7027 04:16:46,337 --> 04:16:47,905 CALLED PATH FROM THE BENCH TO 7028 04:16:47,905 --> 04:16:48,973 COMPANY AND CLINIC. 7029 04:16:48,973 --> 04:16:51,041 THIS IS REALLY SORT OF A 7030 04:16:51,041 --> 04:16:52,609 BRIDGING SESSION THAT WILL COVER 7031 04:16:52,609 --> 04:16:55,646 TOPICS FROM TUESDAY'S SESSION 7032 04:16:55,646 --> 04:16:57,247 TWO, PARTNERING WITH REGULATORS 7033 04:16:57,247 --> 04:16:59,016 FOR SUCCESS AND SESSION THREE 7034 04:16:59,016 --> 04:17:01,952 DEFINING THE TARGET PROFILE, SO 7035 04:17:01,952 --> 04:17:04,788 HERE WE'LL SORT OF DISCUSS THE 7036 04:17:04,788 --> 04:17:06,890 MORE MATURE INNOVATION IDEAS 7037 04:17:06,890 --> 04:17:09,193 THAT HAVE SORT OF BRIDGED OUT OF 7038 04:17:09,193 --> 04:17:10,828 THE CONCEPT OUT OF THE LAB, 7039 04:17:10,828 --> 04:17:13,097 MAYBE INTO SMALL BUSINESS OR ON 7040 04:17:13,097 --> 04:17:15,632 TO SOME OF THE NIH GRANT 7041 04:17:15,632 --> 04:17:17,835 MECHANISMS OR EVEN WITH SEED 7042 04:17:17,835 --> 04:17:19,136 FUNDING FROM EITHER VENTURE 7043 04:17:19,136 --> 04:17:20,871 CAPITAL OR FROM OTHER SOURCES, 7044 04:17:20,871 --> 04:17:23,073 AND REALLY THINKING ABOUT THE 7045 04:17:23,073 --> 04:17:24,742 NEEDS THERE IN THAT SPACE TO GET 7046 04:17:24,742 --> 04:17:29,313 TO THE NEXT PLACE WHICH WOULD BE 7047 04:17:29,313 --> 04:17:31,482 FINISHING THOSE IND-ENABLING 7048 04:17:31,482 --> 04:17:32,883 STUDIES AND THEN MOVING INTO 7049 04:17:32,883 --> 04:17:36,954 CLINICAL TRIALS. 7050 04:17:36,954 --> 04:17:39,690 AND THE LAST SESSION TODAY WILL 7051 04:17:39,690 --> 04:17:43,627 COVER THE TOPICS FROM TODAY'S 7052 04:17:43,627 --> 04:17:45,162 SESSION, SESSION FOUR, 7053 04:17:45,162 --> 04:17:47,131 NAVIGATING CLINICAL TRIALS, AND 7054 04:17:47,131 --> 04:17:49,099 SESSION FIVE, SUCCESS STORIES 7055 04:17:49,099 --> 04:17:50,200 FROM THE LUNG AND SLEEP 7056 04:17:50,200 --> 04:17:50,534 COMMUNITY. 7057 04:17:50,534 --> 04:17:52,603 HERE WE REALLY WANT TO DISCUSS 7058 04:17:52,603 --> 04:17:55,105 SOME OF THE TOPICS THAT WERE 7059 04:17:55,105 --> 04:17:57,207 ADDRESSED IN THE FIRST SESSION 7060 04:17:57,207 --> 04:17:59,877 THINKING ABOUT EASE OF ACCESS TO 7061 04:17:59,877 --> 04:18:02,646 CLINICAL TRIALS, HOW YOU'RE 7062 04:18:02,646 --> 04:18:03,981 WORKING WITHIN YOUR ACADEMIC 7063 04:18:03,981 --> 04:18:06,583 INSTITUTION OR EITHER WITH AN 7064 04:18:06,583 --> 04:18:09,219 INDUSTRY PARTNER TO GET YOUR IP 7065 04:18:09,219 --> 04:18:11,021 PROTECTION, WORK ON CONTRACTS 7066 04:18:11,021 --> 04:18:12,756 AND THINGS LIKE TIK WOULD 7067 04:18:12,756 --> 04:18:15,759 HELP FACILITATE ADVANCEMENT OF 7068 04:18:15,759 --> 04:18:16,693 AN INDICATION AND MOVING INTO 7069 04:18:16,693 --> 04:18:19,329 THE IND AND INTO THE CLINICAL 7070 04:18:19,329 --> 04:18:21,398 TRIAL, AS WELL AS ANY OF THE 7071 04:18:21,398 --> 04:18:24,501 BARRIERS THAT WE'VE TALKED ABOUE 7072 04:18:24,501 --> 04:18:26,270 ACTUAL EARLY PHASE CLINICAL 7073 04:18:26,270 --> 04:18:28,105 TRIALS, WHETHER THAT BE PATIENT 7074 04:18:28,105 --> 04:18:31,175 ENGAGEMENT STRATEGIES, CLINICAL 7075 04:18:31,175 --> 04:18:34,478 TRIALS OUTCOMES, YOU KNOW, 7076 04:18:34,478 --> 04:18:36,013 AGAIN, SECURING CAPITAL AND 7077 04:18:36,013 --> 04:18:36,980 OTHER THINGS LIKE THAT. 7078 04:18:36,980 --> 04:18:39,183 SO THAT WILL COVER THAT SESSION. 7079 04:18:39,183 --> 04:18:42,219 AND WE'LL HAVE TWO CO-MODERATORS 7080 04:18:42,219 --> 04:18:43,787 IN THE SESSION THAT WILL REALLY 7081 04:18:43,787 --> 04:18:46,623 HELP G THEISCUSSION, AND 7082 04:18:46,623 --> 04:18:49,026 REALLY, WE REALLY HOPE THAT YOU 7083 04:18:49,026 --> 04:18:51,095 WILL SPREAD YOURSELF OUT EVENLY 7084 04:18:51,095 --> 04:18:53,530 ACROSS THESE DIFFERENT BREAKOUT 7085 04:18:53,530 --> 04:18:55,032 ROOMS AND THE DISCUSSANTS IN 7086 04:18:55,032 --> 04:18:56,834 THERE CAN REALLY START THINKING 7087 04:18:56,834 --> 04:18:59,736 ABOUT WAYS TO MORE OPENLY SHARE 7088 04:18:59,736 --> 04:19:02,806 SOME OF THEIR OWN EXPERIENCES 7089 04:19:02,806 --> 04:19:04,108 AND THE WORKSHOP PLANNING 7090 04:19:04,108 --> 04:19:05,542 COMMITTEE MEMBERS AND 7091 04:19:05,542 --> 04:19:06,510 COMODERATORS WILL HELP 7092 04:19:06,510 --> 04:19:07,945 FACILITATE THAT AND WILL BE 7093 04:19:07,945 --> 04:19:11,215 TAKING SOME NOTES. 7094 04:19:11,215 --> 04:19:13,183 WE'RE GOING TO BE IN THESE 7095 04:19:13,183 --> 04:19:14,384 BREAKOUT ROOMS, WE ORIGINALLY 7096 04:19:14,384 --> 04:19:15,486 SCHEDULED THEM FOR AN HOUR, BUT 7097 04:19:15,486 --> 04:19:17,521 I THINK THATN THE SMALL 7098 04:19:17,521 --> 04:19:19,022 NUMBERS OF INDIVIDUALS WEIVE 7099 04:19:19,022 --> 04:19:20,324 RIGHT NOW AT THE END OF THE DAY, 7100 04:19:20,324 --> 04:19:22,059 THIS IS GOING TO BE A LITTLE 7101 04:19:22,059 --> 04:19:24,895 MORE INTIMATE AND SO WE MAY NOT 7102 04:19:24,895 --> 04:19:26,230 NEED AS MUCH TIME. 7103 04:19:26,230 --> 04:19:27,431 SO I THINK WE'RE GOING TO PLAN 7104 04:19:27,431 --> 04:19:30,934 ON HAVING ABOUT 30 MINUTES IN 7105 04:19:30,934 --> 04:19:32,236 THAT DISCUSSION, AND THEN THE 7106 04:19:32,236 --> 04:19:33,537 MODERATORS CAN LET US KNOW IF WE 7107 04:19:33,537 --> 04:19:35,205 DO NEED ADDITIONAL TIME, BUT 7108 04:19:35,205 --> 04:19:37,441 WE'RE GOING TO CUT DOWN THE 7109 04:19:37,441 --> 04:19:37,908 BREAKOUT SESSION TO 7110 04:19:37,908 --> 04:19:38,475 AES LITTLE 7111 04:19:38,475 --> 04:19:41,278 BIT SHO AND THEN AFTER 7112 04:19:41,278 --> 04:19:43,147 THAT, WE WILLE ANOTHER 7113 04:19:43,147 --> 04:19:43,714 15-MINUTE BREAK. 7114 04:19:43,714 --> 04:19:47,885 THIS WILL ALLOW THE MODERATORS 7115 04:19:47,885 --> 04:19:49,286 TO DISCUSS THEIR NOTES AND THEN 7116 04:19:49,286 --> 04:19:52,222 WE'LL ALL COME BACK TO THIS MAIN 7117 04:19:52,222 --> 04:19:53,557 ROOM WHERE EACH OF THE BREAKOUTS 7118 04:19:53,557 --> 04:19:55,192 CAN REPORT OFF OF THEIR 7119 04:19:55,192 --> 04:19:57,928 DISCUSSION, AND THEN THE 7120 04:19:57,928 --> 04:19:59,663 WORKSHOP CO-CHAIRS WILL SORT OF 7121 04:19:59,663 --> 04:20:01,331 DISCUSS SOME OF THE CRITICAL 7122 04:20:01,331 --> 04:20:03,500 ELEMENTS WE WANT TO MAKE 7123 04:20:03,500 --> 04:20:04,868 RECOMMENDATIONS ON FOR THE 7124 04:20:04,868 --> 04:20:08,105 PULMONARY AND SLEEP RESEARCH 7125 04:20:08,105 --> 04:20:09,306 COMMUNITY, AND THINK ABOUT 7126 04:20:09,306 --> 04:20:11,175 STRATEGIES THAT WE CAN DO 7127 04:20:11,175 --> 04:20:12,809 TOGETHER AS A COMMUNITY TO 7128 04:20:12,809 --> 04:20:13,911 ADVANCE INNOVATIONS TO THE 7129 04:20:13,911 --> 04:20:14,211 CLINIC. 7130 04:20:14,211 --> 04:20:16,780 AND SO THAT'S REALLY GOING TO BE 7131 04:20:16,780 --> 04:20:18,048 THE STRUCTURE, AND SO NOW WE CAN 7132 04:20:18,048 --> 04:20:21,251 GO AHEAD AND OPEN UP THOSE 7133 04:20:21,251 --> 04:20:22,419 BREAKOUT ROOMS. 7134 04:20:22,419 --> 04:20:24,855 IF YOU WANT TO SEE WHAT THOSE 7135 04:20:24,855 --> 04:20:26,156 TITLES ARE AGAIN, THEY ARE IN 7136 04:20:26,156 --> 04:20:27,558 THE AGENDA AND YOU CAN 7137 04:20:27,558 --> 04:20:28,559 SELF-SELECT ANY OF THE BREAKOUT 7138 04:20:28,559 --> 04:20:29,893 ROOMS AND THEN WE'LL GIVE A FEW 7139 04:20:29,893 --> 04:20:30,961 MINUTES FOR PEOPLE TO GET INTO 7140 04:20:30,961 --> 04:20:31,962 THOSE ROOMS AND THEN WE'LL 7141 04:20:31,962 --> 04:20:38,325 BEGIN. 7142 04:20:38,325 --> 04:20:39,526 5 TO 10 MINUTES TO SUM UP THE 7143 04:20:39,526 --> 04:20:40,260 MAIN DISCUSSION POINTS FROM EACH 7144 04:20:40,260 --> 04:20:43,129 OF THE BREAKOUT ROOMS AND THEN 7145 04:20:43,129 --> 04:20:44,865 JEFF AND KATELYN, AS THE 7146 04:20:44,865 --> 04:20:47,167 WORKSHOP CO-CHAIRS, REALLY 7147 04:20:47,167 --> 04:20:48,902 TAKING THOSE IDEAS TO THINK 7148 04:20:48,902 --> 04:20:50,136 ABOUT THE RECOMMENDATIONS THAT 7149 04:20:50,136 --> 04:20:52,005 WE'D LIKE TO MAKE TO THE 7150 04:20:52,005 --> 04:20:53,306 RESEARCH COMMUNITY AND THAT 7151 04:20:53,306 --> 04:20:55,475 INCLUDES AT EVERY LEVEL, WHETHER 7152 04:20:55,475 --> 04:20:58,645 IT BE A PATIENT AND ADVOCACY 7153 04:20:58,645 --> 04:21:01,715 GROUP, CLINICIANS, INDUSTRY, 7154 04:21:01,715 --> 04:21:03,450 REGULATORS, FEDERAL GOVERNMENT, 7155 04:21:03,450 --> 04:21:04,985 WE'RE ALL UNDER THAT UMBRELLA 7156 04:21:04,985 --> 04:21:08,488 FOR CONSIDERING THESE 7157 04:21:08,488 --> 04:21:10,790 RECOMMENDATIONS AS BARRIERS AND 7158 04:21:10,790 --> 04:21:12,859 FACILITATORS TO GETTING THESE 7159 04:21:12,859 --> 04:21:15,161 WONDERFUL IDEAS THAT ARE 7160 04:21:15,161 --> 04:21:17,030 POTENTIAL INTERVENTIONS ALL THE 7161 04:21:17,030 --> 04:21:18,565 WAY THROUGH TO THE PATIENTS THAT 7162 04:21:18,565 --> 04:21:23,803 NEED THEM MOST. 7163 04:21:23,803 --> 04:21:25,238 SO WE'LL START WITH BREAKOUT 7164 04:21:25,238 --> 04:21:32,913 ROOM ONE. 7165 04:21:32,913 --> 04:21:34,981 >> IF THERE WERE TO BE A WINNER, 7166 04:21:34,981 --> 04:21:36,716 WE WOULD BE THE BEST BREAKOUT 7167 04:21:36,716 --> 04:21:38,151 ROOM, I'M JUST GOING TO GO AHEAD 7168 04:21:38,151 --> 04:21:38,785 AND PREFACE WITH THAT. 7169 04:21:38,785 --> 04:21:40,754 >> OH, YEAH, WE'RE BREAKOUT ROOM 7170 04:21:40,754 --> 04:21:42,589 1. 7171 04:21:42,589 --> 04:21:42,756 HEY. 7172 04:21:42,756 --> 04:21:44,391 >> HELLO, JOHN, NICE TO SEE YOU! 7173 04:21:44,391 --> 04:21:47,227 >> GOOD TO SEE YOU GUYS. 7174 04:21:47,227 --> 04:21:48,094 JOHN KENNEDY. 7175 04:21:48,094 --> 04:21:49,629 GOOD TO TALK TO EVERYONE. 7176 04:21:49,629 --> 04:21:51,498 SUCH A FANTASTIC MEETING. 7177 04:21:51,498 --> 04:21:54,534 YESTERDAY WAS AMAZING. 7178 04:21:54,534 --> 04:21:56,202 IN OUR BREAKOUT ROOM, WE HAD 7179 04:21:56,202 --> 04:21:59,372 SOME REALLY GREAT CONVERSATIONS. 7180 04:21:59,372 --> 04:22:01,775 AGAIN, OUR TITLE WAS MODELS AND 7181 04:22:01,775 --> 04:22:03,510 METHODS INCLUDING COMPUTATIONAL 7182 04:22:03,510 --> 04:22:06,780 IN VITRO AND I VEE VO. 7183 04:22:06,780 --> 04:22:07,113 IN IN VIVO. 7184 04:22:07,113 --> 04:22:08,548 I STARTED BY INTRODUCING MYSELF 7185 04:22:08,548 --> 04:22:11,084 IN SOME OF OUR WORK IN PEDIATRIC 7186 04:22:11,084 --> 04:22:11,918 INTERSTITIAL LUNG DISEASE, 7187 04:22:11,918 --> 04:22:17,958 GENERATING A SINGLE RNA SEQ -- 7188 04:22:17,958 --> 04:22:19,593 KIDS WITH GENETIC PEDIATRIC 7189 04:22:19,593 --> 04:22:20,160 INTERSTITIAL LUNG DISEASE. 7190 04:22:20,160 --> 04:22:21,227 I DISCUSSED SOME OF OUR 7191 04:22:21,227 --> 04:22:22,329 CHALLENGES IN DOING THAT. 7192 04:22:22,329 --> 04:22:25,165 AND THEN WE TALKED ABOUT SORT OF 7193 04:22:25,165 --> 04:22:30,003 WHAT THE BIG SINGLE NUKE AND 7194 04:22:30,003 --> 04:22:31,504 SINGLE CELL MODELS THAT WERE OUT 7195 04:22:31,504 --> 04:22:33,440 THERE WERE, WHAT WAS DONE AND 7196 04:22:33,440 --> 04:22:35,241 WHAT WAS NEEDED TO BE DONE. 7197 04:22:35,241 --> 04:22:38,411 WE DISCUSSED A BIT ABOUT THE 7198 04:22:38,411 --> 04:22:39,946 DIFFERENCES BETWEEN SINGLE CELL 7199 04:22:39,946 --> 04:22:41,781 APPROACH AND THE SINGLE NUC 7200 04:22:41,781 --> 04:22:43,316 APPROACH, WHAT THE BENEFITS AND 7201 04:22:43,316 --> 04:22:44,551 DRAW BACKS OF EACH INCLUDING 7202 04:22:44,551 --> 04:22:50,991 WHAT OUR SAMPLE AVAILABILITY IS. 7203 04:22:50,991 --> 04:22:53,627 WE SPENT A BUNCH OF TIME TALKING 7204 04:22:53,627 --> 04:22:55,362 ABOUT A LUNG ON A CHIP MODEL, 7205 04:22:55,362 --> 04:22:57,330 BEEN AROUND FOR A LITTLE WHILE. 7206 04:22:57,330 --> 04:22:59,633 DEFINITELY A BUNCH OF ADVANCES, 7207 04:22:59,633 --> 04:23:03,269 WE SAW SUCH A GREAT PRESENTATION 7208 04:23:03,269 --> 04:23:04,371 YESTERDAY LOOKING AT OUR IMMUNE 7209 04:23:04,371 --> 04:23:04,904 CELLS MOVING. 7210 04:23:04,904 --> 04:23:11,678 WE TALKED ABOUT HOW COMBINING 7211 04:23:11,678 --> 04:23:13,213 EPITHELIAL, MESENCHYMAL, IMMUNE 7212 04:23:13,213 --> 04:23:14,848 AND LYMPHATIC TOGETHER IN THAT 7213 04:23:14,848 --> 04:23:16,282 SETTING IS VALUABLE BUT WE 7214 04:23:16,282 --> 04:23:18,451 TALKED ABOUT EXACTLY WHAT IS THE 7215 04:23:18,451 --> 04:23:20,120 RIGHT MODEL THERE. 7216 04:23:20,120 --> 04:23:23,690 WE TALKED ABOUT IN THE NEED PEDC 7217 04:23:23,690 --> 04:23:24,858 INTERSTITIAL LUNG DISEASE FIELD, 7218 04:23:24,858 --> 04:23:27,160 HOW SOME OF OUR FIBROTIC LUNG 7219 04:23:27,160 --> 04:23:28,828 DISEASES DUE TO IMMUNE 7220 04:23:28,828 --> 04:23:31,331 DYSREGULATION INCLUDING DEFECTS 7221 04:23:31,331 --> 04:23:34,734 IN COPA, DEFECTS IN STAT 7222 04:23:34,734 --> 04:23:36,369 PATHWAYS MIGHT BE NICE THERE FOR 7223 04:23:36,369 --> 04:23:37,904 MODELING THOSE. 7224 04:23:37,904 --> 04:23:40,240 THERE WAS A BIGGER THEME THAT 7225 04:23:40,240 --> 04:23:43,476 CAME OUT OVER AND OVER ABOUT 7226 04:23:43,476 --> 04:23:45,645 WHAT SAMPLES ARE AVAILABLE NOW. 7227 04:23:45,645 --> 04:23:47,947 AS WE TALK MORE AND MORE ABOUT 7228 04:23:47,947 --> 04:23:50,583 OUR PROJECT FOR THE CHILD 7229 04:23:50,583 --> 04:23:53,086 FOUNDATION DOING THE SINGLE NEUK 7230 04:23:53,086 --> 04:23:55,288 RNA SEQ AND THEN SPATIAL 7231 04:23:55,288 --> 04:23:56,923 TRANSCRIPTOMICS ON THE OLD 7232 04:23:56,923 --> 04:24:00,427 TISSUES, WE BROUGHT UP LUNG MAP 7233 04:24:00,427 --> 04:24:04,631 AND LUNG MAP BRIN DELL 7234 04:24:04,631 --> 04:24:08,835 REPOSITORY, WHICH BEAUTIFULLY 7235 04:24:08,835 --> 04:24:11,037 PRESERVES FROZEN EXPLANTS FROM 7236 04:24:11,037 --> 04:24:13,440 CHILDREN, SOME OF WHOM HAVE 7237 04:24:13,440 --> 04:24:15,308 GENETIC SURFACTANT DEFECTS OR 7238 04:24:15,308 --> 04:24:16,609 INTERSTITIAL LUNG DISEASE AND IS 7239 04:24:16,609 --> 04:24:17,610 A RESOURCE THAT'S AVAILABLE TO 7240 04:24:17,610 --> 04:24:21,381 THE COMMUNITY. 7241 04:24:21,381 --> 04:24:24,417 WE USE THAT IN OUR RESEARCH, AND 7242 04:24:24,417 --> 04:24:26,286 WE WERE FIRST OF ALL WANTING TO 7243 04:24:26,286 --> 04:24:27,387 REPLICATE THAT MORE, AND SECOND 7244 04:24:27,387 --> 04:24:28,788 OF ALL, WANTING TO SPREAD THE 7245 04:24:28,788 --> 04:24:31,324 WORD THAT THAT'S A TOOL FOR 7246 04:24:31,324 --> 04:24:33,393 RESEARCHERS AS WELL. 7247 04:24:33,393 --> 04:24:34,928 WE SPENT A LOT OF TIME TALKING 7248 04:24:34,928 --> 04:24:38,098 ABOUT THE COOL STUFF WITH THE 7249 04:24:38,098 --> 04:24:39,966 FERRET MODEL. 7250 04:24:39,966 --> 04:24:49,576 I GEEKED OUT ON THE ABERRANT 7251 04:24:49,576 --> 04:24:51,644 BASALOID POPULATION AND IF ANY 7252 04:24:51,644 --> 04:24:53,513 WORK HAD BEEN DONE IN 7253 04:24:53,513 --> 04:24:54,948 DEVELOPMENTAL STAGES OF THOSE 7254 04:24:54,948 --> 04:24:56,616 FERRETS BECAUSE AS A PEDIATRIC 7255 04:24:56,616 --> 04:25:00,086 FULL PULL MI KNOLL GIST, WE'RE 7256 04:25:00,086 --> 04:25:02,155 INTERESTED IN FIBROSIS THAT 7257 04:25:02,155 --> 04:25:03,423 ARISES VERY EARLY, SOMETIMES IN 7258 04:25:03,423 --> 04:25:06,192 THE FIRST WEEKS OR MONTHS OF 7259 04:25:06,192 --> 04:25:06,392 LIFE. 7260 04:25:06,392 --> 04:25:13,099 SO THAT LED US TO DISCUSSIONS 7261 04:25:13,099 --> 04:25:14,634 ABOUT FATE MAPPING OF DIFFERENT 7262 04:25:14,634 --> 04:25:15,268 LUNG CELL TYPES. 7263 04:25:15,268 --> 04:25:17,036 WHAT HAD BEEN PUBLISHED 7264 04:25:17,036 --> 04:25:18,772 INCLUDING SOME MOUSE WORK THAT 7265 04:25:18,772 --> 04:25:20,406 IS BEAUTIFUL AND WHAT WAS LEFT 7266 04:25:20,406 --> 04:25:24,477 TO BE PUBLISHED OR TO BE 7267 04:25:24,477 --> 04:25:28,414 INVESTIGATED, WE SEE THAT AS 7268 04:25:28,414 --> 04:25:32,352 POTENTIALLY A PRIORITY FOR 7269 04:25:32,352 --> 04:25:33,987 MODELS IN OUR FIELD AND WORTH 7270 04:25:33,987 --> 04:25:37,257 PUTTING SOME FUNDING BEHIND. 7271 04:25:37,257 --> 04:25:41,628 WE ALSO TALKED ABOUT -- WE GOT 7272 04:25:41,628 --> 04:25:42,962 INTO CONVERSATIONS ABOUT THIS 7273 04:25:42,962 --> 04:25:44,030 GREAT PARTNERSHIP THAT HAD 7274 04:25:44,030 --> 04:25:47,767 HAPPENED IN THIS CONFERENCE 7275 04:25:47,767 --> 04:25:50,637 BETWEEN REPRESENTATIVES FROM 7276 04:25:50,637 --> 04:25:51,704 VENTURE CAPITAL AND FROM 7277 04:25:51,704 --> 04:25:53,907 DIFFERENT AREAS OF INDUSTRY, AND 7278 04:25:53,907 --> 04:25:55,775 WE TALKED ABOUT HOW SOME OTHER 7279 04:25:55,775 --> 04:25:58,711 ORGANIZATIONS INCLUDING THE CZI 7280 04:25:58,711 --> 04:26:00,880 DO THAT REGULARLY AND HOW WE 7281 04:26:00,880 --> 04:26:02,549 SHOULD CONTINUE DOING THAT. 7282 04:26:02,549 --> 04:26:06,352 AND THIS LED US TO A 7283 04:26:06,352 --> 04:26:11,157 CONVERSATION OF THE MERITS OF 7284 04:26:11,157 --> 04:26:16,196 NEW DRUG NOVEL COMPOUNDS, 7285 04:26:16,196 --> 04:26:17,630 REPURPOSING COMPOUNDS AS 7286 04:26:17,630 --> 04:26:20,366 PEDIATRIC PROVIDERS, WE HAVE 7287 04:26:20,366 --> 04:26:22,335 EVEN FEWER TOOLS THAN WHAT I'M 7288 04:26:22,335 --> 04:26:24,103 SEEING ADULTS AND SOMETIMES 7289 04:26:24,103 --> 04:26:26,372 WE'LL LOOK TO REPURPOSE DRUGS 7290 04:26:26,372 --> 04:26:26,773 FIRST. 7291 04:26:26,773 --> 04:26:29,342 SO WE WONDERED HOW TO BALANCE 7292 04:26:29,342 --> 04:26:39,052 THAT AS WELL WITH OUR, PHARMA 7293 04:26:39,052 --> 04:26:41,287 AND OUR BC COLLABORATORS AS WELL 7294 04:26:41,287 --> 04:26:44,324 M AND THEN BI BIGGER CONVERSATIS 7295 04:26:44,324 --> 04:26:46,926 ON BIGGER BIOBANKS AND HOW WE 7296 04:26:46,926 --> 04:26:49,729 COULD GET TO THIS BIOMARKER 7297 04:26:49,729 --> 04:26:50,997 PROBLEM THAT WE'RE ALL 7298 04:26:50,997 --> 04:26:52,198 STRUGGLING WITH HOW TO IDENTIFY 7299 04:26:52,198 --> 04:26:53,399 DISEASE AND THEN HOW TO TRACK 7300 04:26:53,399 --> 04:26:54,167 OUR THERAPIES. 7301 04:26:54,167 --> 04:26:55,668 IN OUR CHILD'S MEETING LAST 7302 04:26:55,668 --> 04:26:58,638 MONTH, WE REALLY DISCUSSED 7303 04:26:58,638 --> 04:27:00,273 EXPANDING OUR CHILD REGISTRY TO 7304 04:27:00,273 --> 04:27:01,708 INCLUDE BLOOD SAMPLES AT 7305 04:27:01,708 --> 04:27:03,009 DIFFERENT TIME POINTS SO THAT 7306 04:27:03,009 --> 04:27:05,311 OUR INVESTIGATORS COULD DO DRUG 7307 04:27:05,311 --> 04:27:10,350 DISCOVERY AND DO THERAPY 7308 04:27:10,350 --> 04:27:10,783 MONITORING TOO. 7309 04:27:10,783 --> 04:27:12,552 A BIGGER EFFORT LIKE THAT IS 7310 04:27:12,552 --> 04:27:14,387 OBVIOUSLY HARDER BUT MAYBE 7311 04:27:14,387 --> 04:27:17,257 COMBINING MORE DISEASE-SPECIFIC 7312 04:27:17,257 --> 04:27:20,093 BIOBANKS INTO AT LEAST 7313 04:27:20,093 --> 04:27:21,628 INFORMATION THAT OTHERS COULD 7314 04:27:21,628 --> 04:27:25,365 HAVE ACCESS TO. 7315 04:27:25,365 --> 04:27:27,000 THOSE, I THINK, WERE THE BIG 7316 04:27:27,000 --> 04:27:29,535 THEMES THAT CAME OUT OF OUR 7317 04:27:29,535 --> 04:27:32,038 GROUP. 7318 04:27:32,038 --> 04:27:33,907 ANYONE ELSE HAVE ANYTHING THAT I 7319 04:27:33,907 --> 04:27:36,743 MISSED? 7320 04:27:36,743 --> 04:27:38,611 >> YOU DID A GREAT JOB 7321 04:27:38,611 --> 04:27:39,579 SUMMARIZING EVERYTHING WE TALKED 7322 04:27:39,579 --> 04:27:41,547 ABOUT. 7323 04:27:41,547 --> 04:27:44,751 >> THANKS. 7324 04:27:44,751 --> 04:27:46,019 >> ALL RIGHT, ROB. 7325 04:27:46,019 --> 04:27:47,987 OVER TO YOU, AND I'LL BACK YOU 7326 04:27:47,987 --> 04:27:50,523 UP IF NEEDED. 7327 04:27:50,523 --> 04:27:53,593 >> THANK YOU. 7328 04:27:53,593 --> 04:27:55,094 SO THIS WAS BREAKOUT SESSION TWO 7329 04:27:55,094 --> 04:27:58,398 WHICH IS CLEARLY THE BEST ONE. 7330 04:27:58,398 --> 04:28:01,234 AND THIS IS PATH FROM BENCH TO 7331 04:28:01,234 --> 04:28:02,568 COMPANY AND CLINIC. 7332 04:28:02,568 --> 04:28:05,605 SO JUST TO GIVE YOU HIGH LEVEL 7333 04:28:05,605 --> 04:28:08,541 OVERVIEW, WE FOUND FIVE AREAS 7334 04:28:08,541 --> 04:28:12,278 THAT ARE WORTH COMMENTING ON. 7335 04:28:12,278 --> 04:28:16,749 NUMBER ONE IS JUST HOW DO WE LET 7336 04:28:16,749 --> 04:28:18,084 EARLY STAGE INVESTIGATORS KNOW 7337 04:28:18,084 --> 04:28:19,819 THAT THERE ARE ADDITIONAL 7338 04:28:19,819 --> 04:28:20,920 RESOURCES OUTSIDE OF FEDERAL 7339 04:28:20,920 --> 04:28:22,722 FUNDING WHICH MAY BE SLIGHTLY 7340 04:28:22,722 --> 04:28:25,758 EASIER TO GET AND A LOWER BAR, 7341 04:28:25,758 --> 04:28:27,694 WHICH MIGHT HELP KIND OF FUND 7342 04:28:27,694 --> 04:28:30,229 THE EARLY DISCOVERY BEFORE IT'S 7343 04:28:30,229 --> 04:28:30,997 NIH-READY, WE THINK THAT'S 7344 04:28:30,997 --> 04:28:31,297 IMPORTANT. 7345 04:28:31,297 --> 04:28:33,499 AND THEN HOW CAN THE NIH START 7346 04:28:33,499 --> 04:28:34,934 TO EDUCATE PEOPLE ABOUT THAT. 7347 04:28:34,934 --> 04:28:37,203 SO FOR EXAMPLE, WHETHER IT'S THE 7348 04:28:37,203 --> 04:28:39,472 CF FOUNDATION, LIKE LOCAL SEED 7349 04:28:39,472 --> 04:28:41,341 FUNDING WHICH IS AT THE STATE OR 7350 04:28:41,341 --> 04:28:44,077 REGIONAL LEVEL OR BIOTECH HUBS, 7351 04:28:44,077 --> 04:28:47,580 FOR EXAMPLE, THEY HAVE THE NORTH 7352 04:28:47,580 --> 04:28:51,184 CAROLINA BIOTECH CENTER, BECAUSE 7353 04:28:51,184 --> 04:28:52,952 IT'S REGIONAL IT'S LIKELY THEY 7354 04:28:52,952 --> 04:28:54,687 HAVE LESS COMPETITIVENESS, A 7355 04:28:54,687 --> 04:28:56,456 LITTLE EASIER TO KIND OF DO THAT 7356 04:28:56,456 --> 04:28:58,291 AND THEN MAYBE THEY'LL BE 7357 04:28:58,291 --> 04:29:00,059 WILLING TO TAKE ON SOME MORE 7358 04:29:00,059 --> 04:29:00,626 RISK. 7359 04:29:00,626 --> 04:29:04,197 NUMBER TWO, THIS IS REALLY 7360 04:29:04,197 --> 04:29:06,399 GETTING STARTED IN DRUG 7361 04:29:06,399 --> 04:29:07,166 DISCOVERY AND KIND OF GETTING 7362 04:29:07,166 --> 04:29:08,568 OFF THE BENCH BEFORE WE EVEN GET 7363 04:29:08,568 --> 04:29:09,469 TO THE COMPANY. 7364 04:29:09,469 --> 04:29:10,970 SO THEN THE OTHER ONE IS 7365 04:29:10,970 --> 04:29:12,505 CREATIVE GROUND MECHANISMS. 7366 04:29:12,505 --> 04:29:17,977 WE'VE GOT THE R01s AND R21s, 7367 04:29:17,977 --> 04:29:18,978 ONCE YOU FORM THE COMPANY YOU 7368 04:29:18,978 --> 04:29:21,814 CAN DO THE STTRs AND SBRs 7369 04:29:21,814 --> 04:29:23,683 AND IN THE MIDDLE THERE'S STUFF 7370 04:29:23,683 --> 04:29:24,017 LACKING. 7371 04:29:24,017 --> 04:29:26,753 SO THERE COULD BE ONE -- IT'S 7372 04:29:26,753 --> 04:29:28,488 ALMOST LIKE AN STTR AT THE 7373 04:29:28,488 --> 04:29:29,789 UNIVERSITY BUT FEDERALLY THEY 7374 04:29:29,789 --> 04:29:31,424 CAN'T DO THAT SO WE'D HAVE TO 7375 04:29:31,424 --> 04:29:34,160 HAVE A NEW MECHANISM. 7376 04:29:34,160 --> 04:29:36,029 AND SOMETHING WHICH MAY HELP, 7377 04:29:36,029 --> 04:29:38,965 FOR EXAMPLE, IF YOU HAVE A DRUG 7378 04:29:38,965 --> 04:29:40,199 THAT WANTS TO BE REPURPOSED, 7379 04:29:40,199 --> 04:29:43,469 IT'S NOT NOVEL ENOUGH FOR AN 7380 04:29:43,469 --> 04:29:46,773 R01, IT COULD STILL EXTREMELY 7381 04:29:46,773 --> 04:29:48,074 VALUABLE AND MAY ACTUALLY GET TO 7382 04:29:48,074 --> 04:29:48,841 THE CLINIC. 7383 04:29:48,841 --> 04:29:50,143 SO THINGS LIKE THAT. 7384 04:29:50,143 --> 04:29:55,081 POINT NUMBER THREE WAS ARE 7385 04:29:55,081 --> 04:29:57,784 INSTITUTIONS A BARRIER TO DRUG 7386 04:29:57,784 --> 04:30:01,654 DEVELOPMENT AND THIS IS BOTH THE 7387 04:30:01,654 --> 04:30:04,023 ACADEMIC INSTITUTES OR THE NIH, 7388 04:30:04,023 --> 04:30:05,992 MAYBE IP IS NOT FULLY PROTECTED, 7389 04:30:05,992 --> 04:30:07,760 THERE COULD BE A BARRIER TO 7390 04:30:07,760 --> 04:30:10,363 PROGRESS, AGAIN, BOTH 7391 04:30:10,363 --> 04:30:14,100 INSTITUTIONAL -- IS TO -- DOES 7392 04:30:14,100 --> 04:30:15,968 IT IMPEACH -- CAN THAT BE LOOKED 7393 04:30:15,968 --> 04:30:21,541 AT, CAN THE NIH GUIDE THE INTS 7394 04:30:21,541 --> 04:30:22,675 INSTITUTIONS BETTER. 7395 04:30:22,675 --> 04:30:23,843 I THINK THERE WAS ALSO A 7396 04:30:23,843 --> 04:30:25,144 DISCUSSION ABOUT THE CULTURE AT 7397 04:30:25,144 --> 04:30:26,012 THE INSTITUTIONAL LEVEL. 7398 04:30:26,012 --> 04:30:28,414 SO FOR EXAMPLE, ACADEMIC 7399 04:30:28,414 --> 04:30:32,018 INDUSTRY, TRULY ARE THE R01s 7400 04:30:32,018 --> 04:30:33,252 AND -- THEN OBVIOUSLY LESS VALUE 7401 04:30:33,252 --> 04:30:35,421 IS PLACED ON INVESTIGATORS 7402 04:30:35,421 --> 04:30:36,956 GETTING DRUG DEVELOPMENT 7403 04:30:36,956 --> 04:30:37,190 FUNDING. 7404 04:30:37,190 --> 04:30:38,491 THERE COULD BE SOMETHING, 7405 04:30:38,491 --> 04:30:40,460 HOWEVER, THE DRUG DEVELOPMENT IS 7406 04:30:40,460 --> 04:30:43,129 ACTUALLY CENTRAL TOLL THE NIH'S 7407 04:30:43,129 --> 04:30:44,931 MISSION STATEMENT, SO COULD THE 7408 04:30:44,931 --> 04:30:46,899 NIH EITHER BY EDUCATING KIND OF 7409 04:30:46,899 --> 04:30:54,674 THE SENIOR ACADEMICS OR 7410 04:30:54,674 --> 04:30:58,277 INCENTIVIZING, SO THE BASIC 7411 04:30:58,277 --> 04:31:00,012 DISCOVERY TRACK -- MAYBE THE 7412 04:31:00,012 --> 04:31:03,116 ENTREPRENEUR TRACK OR THE 7413 04:31:03,116 --> 04:31:07,019 TRANSLATIONAL TRACK IS MORE 7414 04:31:07,019 --> 04:31:08,254 VALUABLE, THIS IS SOMETHING THAT 7415 04:31:08,254 --> 04:31:10,323 COULD BE AFFECTED. 7416 04:31:10,323 --> 04:31:12,058 AND SO THE QUESTION IS ALSO HOW 7417 04:31:12,058 --> 04:31:15,695 WOULD THE NIH DO THIS, BY 7418 04:31:15,695 --> 04:31:16,996 COMMUNICATION, TO MAKE THE 7419 04:31:16,996 --> 04:31:18,097 ACADEMICS AWARE THAT THE DRUG 7420 04:31:18,097 --> 04:31:19,298 DEVELOPMENT IS CENTRAL TO THEIR 7421 04:31:19,298 --> 04:31:21,033 MISSION STATEMENT, OR BY 7422 04:31:21,033 --> 04:31:25,071 SHIFTING RESOURCES. 7423 04:31:25,071 --> 04:31:28,040 AND THEN CAN THE NIH PUT IN MORE 7424 04:31:28,040 --> 04:31:29,509 TRANSLATION INVESTMENT BASICALLY 7425 04:31:29,509 --> 04:31:30,543 TO DO THIS. 7426 04:31:30,543 --> 04:31:32,345 SO POINT NUMBER FOUR IS, IT WAS 7427 04:31:32,345 --> 04:31:33,279 DEBATED -- SO YOU KNOW, COULD 7428 04:31:33,279 --> 04:31:36,115 THERE BE A NEW TYPE OF R01 SUCH 7429 04:31:36,115 --> 04:31:40,386 AS A TRANSLATIONAL R01 WHICH WAS 7430 04:31:40,386 --> 04:31:42,355 REALLY FOCUSED ON DRUG 7431 04:31:42,355 --> 04:31:44,590 DEVELOPMENT, BUT TO GET TO THE 7432 04:31:44,590 --> 04:31:49,462 TARGET SO YOU CAN GET GET 7433 04:31:49,462 --> 04:31:53,533 INVESTIGATORS OUT INTO THE OF TE 7434 04:31:53,533 --> 04:31:54,333 INDUSTRY AND INTO THE COMPANY. 7435 04:31:54,333 --> 04:31:56,102 I FOUNDED TWO COMPANIES, ONE GOT 7436 04:31:56,102 --> 04:31:58,838 TO PHASE TWO, ONE IS IN 7437 04:31:58,838 --> 04:32:02,875 PRE-CLINICAL DEVELOPMENT. 7438 04:32:02,875 --> 04:32:04,677 THE FIRST, I RAISED MONEY AND 7439 04:32:04,677 --> 04:32:05,745 LOST CONTROL OF THE COMPANY 7440 04:32:05,745 --> 04:32:06,913 QUITE QUICKLY. 7441 04:32:06,913 --> 04:32:09,882 THE SECOND I'VE RAISED SEVERAL 7442 04:32:09,882 --> 04:32:11,217 MILLION, IT'S GONE MUCH SLOWLY 7443 04:32:11,217 --> 04:32:15,788 BUT I THINK WE'RE IN MUCH 7444 04:32:15,788 --> 04:32:18,090 BETTER -- YOU MEAN, I'M A BIG -- 7445 04:32:18,090 --> 04:32:20,493 I THINK MICHAEL BOLTSMAN -- I 7446 04:32:20,493 --> 04:32:22,895 WAS A BIG ADVOCATE FOR USING THE 7447 04:32:22,895 --> 04:32:24,163 FEDERAL DOLLARS TO GET AS 7448 04:32:24,163 --> 04:32:25,731 PREPARED AS YOU CAN BEFORE YOU 7449 04:32:25,731 --> 04:32:26,832 PARTNER UP BECAUSE I BELIEVE 7450 04:32:26,832 --> 04:32:28,167 THERE'S A LOT YOU CAN DO. 7451 04:32:28,167 --> 04:32:32,104 FOR EXAMPLE, TRANSLATION R01s 7452 04:32:32,104 --> 04:32:33,472 OR NEW FUNDING MECHANISMS COULD 7453 04:32:33,472 --> 04:32:37,143 BE REALLY HELPFUL FOR 7454 04:32:37,143 --> 04:32:37,476 INVESTIGATORS. 7455 04:32:37,476 --> 04:32:42,481 AND THEN THE LAST POINT WAS 7456 04:32:42,481 --> 04:32:44,350 ESPECIALLY THIS VENTURE 7457 04:32:44,350 --> 04:32:47,386 CAPITALISTS HARD TO SECURE, AND 7458 04:32:47,386 --> 04:32:50,890 SO THE NIH HAD A GREAT ROLE IN 7459 04:32:50,890 --> 04:32:52,892 HELPING PEOPLE GO OUT TO VCs. 7460 04:32:52,892 --> 04:32:54,293 FOR EXAMPLE, I KNOW THERE ARE 7461 04:32:54,293 --> 04:32:57,897 THINGS LIKE THE VENTURE SUMMIT, 7462 04:32:57,897 --> 04:33:01,734 LIKE ATS AND THINGS, BUT THERE'S 7463 04:33:01,734 --> 04:33:06,539 NO ONE SIZE FITS ALL SO -- ASK 7464 04:33:06,539 --> 04:33:08,507 PEOPLE ARE THEY READY TO GO OUT, 7465 04:33:08,507 --> 04:33:12,645 IS IT TOO SOON, AND REALLY HELP 7466 04:33:12,645 --> 04:33:13,980 PEOPLE GET READY. 7467 04:33:13,980 --> 04:33:16,816 SO THAT'S ALL I HAVE. 7468 04:33:16,816 --> 04:33:18,884 MARRAH ACTUALLY MADE MUCH MORE 7469 04:33:18,884 --> 04:33:19,552 EXTENSIVE NOTE. 7470 04:33:19,552 --> 04:33:21,387 I CHOSE NOT TO USE THEM AT THIS 7471 04:33:21,387 --> 04:33:22,188 POINT IN TIME. 7472 04:33:22,188 --> 04:33:24,457 I CAN GO BACK TO THEM OR 7473 04:33:24,457 --> 04:33:25,324 ANYTHING ELSE TO ADD? 7474 04:33:25,324 --> 04:33:26,892 >> NO, I THINK YOU COVERED IT. 7475 04:33:26,892 --> 04:33:29,495 I THINK A COUPLE OF OTHER THINGS 7476 04:33:29,495 --> 04:33:31,163 THAT WERE ANCILLARY TO SOME OF 7477 04:33:31,163 --> 04:33:34,700 THAT IS THE IDEA OF DECISION 7478 04:33:34,700 --> 04:33:34,900 POINT. 7479 04:33:34,900 --> 04:33:36,235 YOU TOUCHED ON THIS ABOUT, YOU 7480 04:33:36,235 --> 04:33:38,037 KNOW, LOSING COMPANY RIGHTS 7481 04:33:38,037 --> 04:33:41,641 VERSUS STAYING IN, AND WHETHER 7482 04:33:41,641 --> 04:33:42,975 OR NOT YOU WANT TO FOLLOW YOUR 7483 04:33:42,975 --> 04:33:44,443 SCIENCE ALL THE WAY THROUGH TO 7484 04:33:44,443 --> 04:33:46,145 THE END OR IF YOU WANT TO LET 7485 04:33:46,145 --> 04:33:47,947 GO, BECAUSE THAT'S A BETTER 7486 04:33:47,947 --> 04:33:51,817 OPPORTUNITY FISCALLY AND FOR THE 7487 04:33:51,817 --> 04:33:52,485 INTERVENTION ITSELF. 7488 04:33:52,485 --> 04:33:56,522 I THINK THAT'S A CRITICAL 7489 04:33:56,522 --> 04:33:57,823 QUESTION. 7490 04:33:57,823 --> 04:33:59,792 MIKE TOUCHED ON THIS AS WELL, 7491 04:33:59,792 --> 04:34:01,227 YOUR PERSEVERANCE AND POINT OF 7492 04:34:01,227 --> 04:34:03,529 ABILITY TO CONTINUE TO LEVERAGE 7493 04:34:03,529 --> 04:34:06,799 FEDERAL GOVERNMENT FUNDING AS 7494 04:34:06,799 --> 04:34:08,768 SECURE A RESOURCE TO STAY IN THE 7495 04:34:08,768 --> 04:34:15,341 SPACE BUT I THINK YOU VERY WEREY 7496 04:34:15,341 --> 04:34:19,045 ELOQUENT IN SAYING THAT IS SLOW 7497 04:34:19,045 --> 04:34:21,013 BECAUSE IT'S NOT THE SAME AS 7498 04:34:21,013 --> 04:34:21,914 RAISING VENTURE CAPITAL FUNDS 7499 04:34:21,914 --> 04:34:23,983 AND GETTING A LARGER AMOUNT OF 7500 04:34:23,983 --> 04:34:25,384 SEED MONEY INTO MOVING THINGS 7501 04:34:25,384 --> 04:34:25,785 ALONG. 7502 04:34:25,785 --> 04:34:27,586 SO THE BALANCE, I THINK, IS 7503 04:34:27,586 --> 04:34:28,688 SOMETHING THAT PEOPLE ARE STILL 7504 04:34:28,688 --> 04:34:30,222 THINKING ABOUT, AND HOW WE CAN 7505 04:34:30,222 --> 04:34:35,561 CONTINUE TO SUPPORT THEM BY 7506 04:34:35,561 --> 04:34:37,229 FINDING WAYS TO GET THEM TO SPIN 7507 04:34:37,229 --> 04:34:38,531 OFF TO DIFFERENT RESOURCES, I 7508 04:34:38,531 --> 04:34:43,135 THINK WAS A KEY THEME. 7509 04:34:43,135 --> 04:34:45,204 >> OKAYMENT WELL, I GUESS WE'LL 7510 04:34:45,204 --> 04:34:46,706 TRANSITION TO THE BEST FOR LAST 7511 04:34:46,706 --> 04:34:48,874 HERE, WITH THE THIRD GROUP, 7512 04:34:48,874 --> 04:34:50,710 WHICH WAS NAVIGATING CLINICAL 7513 04:34:50,710 --> 04:34:54,613 TRIALS AND PARTNERING PATIENTS. 7514 04:34:54,613 --> 04:34:56,482 I WOULD SAY THERE WAS REALLY 7515 04:34:56,482 --> 04:34:57,883 FOUR OVERALL MAIN ARCHING TOPICS 7516 04:34:57,883 --> 04:34:59,285 THAT WE TALKED ABOUT IN OUR 7517 04:34:59,285 --> 04:34:59,618 BREAKOUT. 7518 04:34:59,618 --> 04:35:01,620 I THINK THE FIRST ONE THAT 7519 04:35:01,620 --> 04:35:03,456 REALLY GENERATED A LOT OF 7520 04:35:03,456 --> 04:35:04,557 INTEREST WAS AROUND THE 7521 04:35:04,557 --> 04:35:05,991 POTENTIAL IMPORTANCE AND 7522 04:35:05,991 --> 04:35:07,626 OPPORTUNITY WITH INVESTIGATOR 7523 04:35:07,626 --> 04:35:11,197 INITIATED TRIALS. 7524 04:35:11,197 --> 04:35:13,099 THEY'RE SO PRAGMATIC, THERE'S 7525 04:35:13,099 --> 04:35:14,166 THE OPPORTUNITY FOR ACADEMIC 7526 04:35:14,166 --> 04:35:15,368 MEDICINE TO REALLY TAKE THE LEAD 7527 04:35:15,368 --> 04:35:17,169 ON A LOT OF THESE AND HELP 7528 04:35:17,169 --> 04:35:18,204 HOPEFULLY WITH SUPPORT FROM THE 7529 04:35:18,204 --> 04:35:20,606 NIH TO FINANCE THEM, BUT WE 7530 04:35:20,606 --> 04:35:22,808 CAN'T REALLY UNDERSCORE ENOUGH 7531 04:35:22,808 --> 04:35:24,677 THE IMPORTANCE OF THIS INITIAL 7532 04:35:24,677 --> 04:35:27,079 CLINICAL PROOF OF CONCEPT. 7533 04:35:27,079 --> 04:35:29,382 HOW YOU CAN TAKE A HANDFUL OF 7534 04:35:29,382 --> 04:35:31,784 PATIENTS, NOT THIS MORE 7535 04:35:31,784 --> 04:35:36,822 TRADITIONAL VERY EXTENSIVE 7536 04:35:36,822 --> 04:35:38,457 LARGER STUDY DEPENDING ON THE 7537 04:35:38,457 --> 04:35:42,261 PATIENT POPULATION, BUT THAT 7538 04:35:42,261 --> 04:35:46,432 INITIAL VERY EXPENSIVE LARGER 7539 04:35:46,432 --> 04:35:48,100 STUDY, TO REALLY INCENTIVIZE 7540 04:35:48,100 --> 04:35:50,035 GOING AFTER THESE VERY NICHE 7541 04:35:50,035 --> 04:35:51,470 SMALL PATIENT POPULATIONS AND IT 7542 04:35:51,470 --> 04:35:55,408 REALLY ALSO ADDRESSES THE ACCESS 7543 04:35:55,408 --> 04:35:56,842 PROBLEM FOR PATIENTS BECAUSE A 7544 04:35:56,842 --> 04:35:59,879 LOT OF TIMES IT'S THESE ACADEMIC 7545 04:35:59,879 --> 04:36:01,414 MEDICINE CLINICIANS WHO ARE 7546 04:36:01,414 --> 04:36:02,515 WORKING WITH THOSE PATIENTS IN 7547 04:36:02,515 --> 04:36:05,017 THEIR PRIVATE CLINICS AND CAN 7548 04:36:05,017 --> 04:36:06,886 ACTUALLY BRING THEM FORWARD IN A 7549 04:36:06,886 --> 04:36:07,887 VERY SUCCINCT MANNER. 7550 04:36:07,887 --> 04:36:10,923 SO WE THINK THERE'S A REAL 7551 04:36:10,923 --> 04:36:11,791 OPPORTUNITY THERE TO 7552 04:36:11,791 --> 04:36:13,793 PARTICULARLY FOR SOME OF THE 7553 04:36:13,793 --> 04:36:15,861 RARER LUNG DISEASES, TO REALLY 7554 04:36:15,861 --> 04:36:17,263 MAKE HUGE IMPROVEMENTS IN THE 7555 04:36:17,263 --> 04:36:19,899 SPACE AND LOWER THOSE BARRIERS 7556 04:36:19,899 --> 04:36:22,468 FOR CLINICAL DEVELOPMENT. 7557 04:36:22,468 --> 04:36:23,302 SO THAT WAS CERTAINLY ONE OF THE 7558 04:36:23,302 --> 04:36:25,704 BIG AREAS OF INTEREST. 7559 04:36:25,704 --> 04:36:27,339 I THINK ANOTHER BIG THEME WAS 7560 04:36:27,339 --> 04:36:29,208 REALLY TO HELP BETTER DEFINE THE 7561 04:36:29,208 --> 04:36:30,609 PATIENT POPULATION. 7562 04:36:30,609 --> 04:36:33,045 YOU KNOW, REALLY JUST THE ACCESS 7563 04:36:33,045 --> 04:36:35,014 TO ELECTRONIC MEDICAL RECORDS. 7564 04:36:35,014 --> 04:36:38,918 WE HAD HEARD AROUND THE PCB 7565 04:36:38,918 --> 04:36:41,554 FOUNDATION SOME OF THE WORK THAT 7566 04:36:41,554 --> 04:36:44,623 THEY HAD DONE TO TRY AND GAIN 7567 04:36:44,623 --> 04:36:48,227 ACCESS AND HOW CHALLENGING THAT 7568 04:36:48,227 --> 04:36:49,895 COULD BE, AND EVEN THEN WHEN YOU 7569 04:36:49,895 --> 04:36:51,197 DO GET ACCESS SOMETIMES TO BE 7570 04:36:51,197 --> 04:36:54,066 ABLE TO ACTUALLY PARSE OUT 7571 04:36:54,066 --> 04:36:55,534 ENOUGH INFORMATION TO ACTUALLY 7572 04:36:55,534 --> 04:36:56,135 DO SOMETHING WITH THAT 7573 04:36:56,135 --> 04:36:56,902 INFORMATION IS QUITE 7574 04:36:56,902 --> 04:36:57,236 CHALLENGING. 7575 04:36:57,236 --> 04:36:59,305 SO ANYTHING THAT CAN BE DONE TO 7576 04:36:59,305 --> 04:37:01,273 REALLY EDUCATE PATIENTS AND 7577 04:37:01,273 --> 04:37:02,241 REALLY RECRUIT THEM AS AN 7578 04:37:02,241 --> 04:37:03,476 OPPORTUNITY TO REALLY LEARN, 7579 04:37:03,476 --> 04:37:05,978 LEARN FROM THEIR MEDICAL 7580 04:37:05,978 --> 04:37:08,414 RECORDS, AND THIS ALSO COULD 7581 04:37:08,414 --> 04:37:09,482 POTENTIALLY UNLIKE BETTER 7582 04:37:09,482 --> 04:37:11,016 UNDERSTANDING OF THESE DISEASES, 7583 04:37:11,016 --> 04:37:14,420 WHICH THEN CAN -- YOU HAVE 7584 04:37:14,420 --> 04:37:15,955 SOMETHING LIKE IPF WAS ONE OF 7585 04:37:15,955 --> 04:37:18,457 THE GREAT EXAMPLES THAT WAS 7586 04:37:18,457 --> 04:37:19,992 RAISED, IDIOPATHIC IN ITS NAME, 7587 04:37:19,992 --> 04:37:21,727 IT JUST DOESN'T GENERATE A LOT 7588 04:37:21,727 --> 04:37:23,796 OF UNDERSTANDING AROUND THE 7589 04:37:23,796 --> 04:37:26,632 POTENTIAL FOR VERY SPECIFIC 7590 04:37:26,632 --> 04:37:27,800 GENETIC-BASED SUBPOPULATIONS 7591 04:37:27,800 --> 04:37:30,803 PERHAPS WITHIN THAT LARGER FIELD 7592 04:37:30,803 --> 04:37:33,539 OF THE DISEASE TO REALLY TARGET 7593 04:37:33,539 --> 04:37:34,640 IN ON. 7594 04:37:34,640 --> 04:37:37,910 SO THOSE ARE REALLY OPPORTUNITY. 7595 04:37:37,910 --> 04:37:39,111 THE POTENTIAL IMPORTANCE OF 7596 04:37:39,111 --> 04:37:40,880 NATURAL HISTORY STUDIES, YOU 7597 04:37:40,880 --> 04:37:42,848 KNOW, THOSE ARE THINGS THAT CAN 7598 04:37:42,848 --> 04:37:44,250 REALLY ATTRACT INDUSTRY 7599 04:37:44,250 --> 04:37:46,452 INVESTMENT IF THERE ARE 7600 04:37:46,452 --> 04:37:47,553 WELL-UNDERSTOOD PATIENT 7601 04:37:47,553 --> 04:37:49,188 POPULATIONS AND DISEASE 7602 04:37:49,188 --> 04:37:50,389 PROGRESSION, AND THOSE ARE KIND 7603 04:37:50,389 --> 04:37:52,358 OF THE SAFE HARBOR SPACE, IF YOU 7604 04:37:52,358 --> 04:37:54,226 WILL, THAT BOTH GOVERNMENT AND 7605 04:37:54,226 --> 04:37:56,962 ACADEMIC MEDICINE CAN HELP 7606 04:37:56,962 --> 04:37:59,698 PARTNER ON, WHERE IT'S REALLY 7607 04:37:59,698 --> 04:38:08,107 CHALLENGING IF -- WHEN INDUSTRY 7608 04:38:08,107 --> 04:38:10,843 HAS TO ALSO DEVELOP A NATURAL 7609 04:38:10,843 --> 04:38:12,912 HISTORY STUDY IN PARALLEL TO A 7610 04:38:12,912 --> 04:38:14,313 THERAPEUTIC AND IT'S ALMOST A 7611 04:38:14,313 --> 04:38:15,548 CONCURRENT DOUBLING OF EXPENSE 7612 04:38:15,548 --> 04:38:17,283 IF YOU WILL, SO IT FURTHER 7613 04:38:17,283 --> 04:38:18,617 RAISES THAT BARRIER PARTICULARLY 7614 04:38:18,617 --> 04:38:19,919 FOR RARE DISEASES TO MAKE IT 7615 04:38:19,919 --> 04:38:21,353 THAT MUCH MORE CHALLENGING, 7616 04:38:21,353 --> 04:38:22,555 WHEREAS THIS IS AN OPPORTUNITY, 7617 04:38:22,555 --> 04:38:24,089 PERHAPS, FOR ACADEMIC MEDICINE 7618 04:38:24,089 --> 04:38:25,624 OR THE GOVERNMENT TO START 7619 04:38:25,624 --> 04:38:27,026 REALLY UNDERSTANDING WHAT 7620 04:38:27,026 --> 04:38:31,397 DISEASE PROGRESSION LOOKS LIKE, 7621 04:38:31,397 --> 04:38:37,770 WHICH INEVITABLY INVITES MORE 7622 04:38:37,770 --> 04:38:38,504 RESEARCH BECAUSE AGAIN IT'S 7623 04:38:38,504 --> 04:38:40,506 AROUND LOWERING THOSE BARRIERS 7624 04:38:40,506 --> 04:38:41,574 AND IMPROVING THE CHANCE OF 7625 04:38:41,574 --> 04:38:42,575 SUCCESS WHERE WE DON'T HAVE TO 7626 04:38:42,575 --> 04:38:43,809 TRY AND FIGURE OUT THE 7627 04:38:43,809 --> 04:38:46,645 BIOMARKERS AS WE'RE ROLLING AND 7628 04:38:46,645 --> 04:38:47,413 WHAT SUCCESS LOOKS LIKE IN A 7629 04:38:47,413 --> 04:38:49,481 PATIENT POPULATION. SO WE THINK 7630 04:38:49,481 --> 04:38:50,950 THERE'S REAL OPPORTUNITY THERE. 7631 04:38:50,950 --> 04:38:52,885 WITHIN THE NATURAL HISTORY 7632 04:38:52,885 --> 04:38:55,721 STUDY, THE PCD GROUP ACTUALLY 7633 04:38:55,721 --> 04:38:58,791 ALSO SPOKE ABOUT THE IMPORTANCE 7634 04:38:58,791 --> 04:39:01,627 POF UNDERSTANDING THE ENTIRE 7635 04:39:01,627 --> 04:39:02,728 PATIENT POPULATION WHERE 7636 04:39:02,728 --> 04:39:04,463 POSSIBLE BECAUSE THEY HAD A LOT 7637 04:39:04,463 --> 04:39:06,999 OF GOOD GRAN ALREADY 7638 04:39:06,999 --> 04:39:08,200 UNDERSTANDING AROUND PEDIATRIC 7639 04:39:08,200 --> 04:39:09,501 PATIENTS BUT THEY'VE REALLY 7640 04:39:09,501 --> 04:39:11,036 STRUGGLED TO KIND OF BACKFILL 7641 04:39:11,036 --> 04:39:12,905 WITH WHAT DOES THAT LOOK LIKE IN 7642 04:39:12,905 --> 04:39:13,238 ADULTS? 7643 04:39:13,238 --> 04:39:15,741 AND THAT'S A GOOD LESSON TO 7644 04:39:15,741 --> 04:39:16,842 REALLY UNDERSTAND THAT IF YOU 7645 04:39:16,842 --> 04:39:19,445 FOCUS IN TOO MUCH INTENTIONALLY 7646 04:39:19,445 --> 04:39:23,182 BUT TOO MUCH ON A NICHE 7647 04:39:23,182 --> 04:39:24,049 SUBPOPULATION THAT CAN HELP YOU 7648 04:39:24,049 --> 04:39:25,451 IN SOME REGARDS BUT IT CAN ALSO 7649 04:39:25,451 --> 04:39:29,288 HURT YOU IN OTHERS. 7650 04:39:29,288 --> 04:39:33,125 AS WE HAD TALKED AROUND THE LAST 7651 04:39:33,125 --> 04:39:34,560 SESSIONS, PANEL DISCUSSION WAS 7652 04:39:34,560 --> 04:39:39,131 AROUND THE FOLKS HAVE BEEN TALK, 7653 04:39:39,131 --> 04:39:43,969 ABOUT, JUST REALLY THE 7654 04:39:43,969 --> 04:39:44,970 IMPORTANCE OF THERE REALLY 7655 04:39:44,970 --> 04:39:48,007 TRYING TO DEVELOP PHYSIOLOGICAL 7656 04:39:48,007 --> 04:39:49,408 BASED BIOMARKERS TO COMPLEMENT 7657 04:39:49,408 --> 04:39:51,377 MORE TRADITIONAL BIOMEDICAL OR 7658 04:39:51,377 --> 04:39:52,578 BIOLOGICAL BIOMARKERS OR THINGS 7659 04:39:52,578 --> 04:39:53,812 THAT ARE VERY QUANTIFIABLE. 7660 04:39:53,812 --> 04:39:56,882 BUT NOT ALL BIOMARKERS PRECEDE D 7661 04:39:56,882 --> 04:39:58,717 IN A LINEAR FASHION, SO THAT 7662 04:39:58,717 --> 04:39:59,718 MIGHT BE AN OPPORTUNITY TO AGAIN 7663 04:39:59,718 --> 04:40:01,353 AS PART OF SAY A NATURAL HISTORY 7664 04:40:01,353 --> 04:40:02,554 STUDY INVESTING MORE IN TRYING 7665 04:40:02,554 --> 04:40:06,158 TO UNDERSTAND HOW DIFFERENT 7666 04:40:06,158 --> 04:40:07,860 BIOMARKERS REACT, COUGH MONITOR, 7667 04:40:07,860 --> 04:40:08,994 REMOTE TYPE OF MONITORING OF 7668 04:40:08,994 --> 04:40:12,064 THESE END POINTS AS WE'VE HEARD 7669 04:40:12,064 --> 04:40:12,297 ABOUT. 7670 04:40:12,297 --> 04:40:13,932 THERE COULD BE A LOT OF VALUE IN 7671 04:40:13,932 --> 04:40:17,736 THIS SPACE, AND THAT, AGAIN, 7672 04:40:17,736 --> 04:40:19,371 LOWERS THE ACTIVATION ENERGY, IF 7673 04:40:19,371 --> 04:40:21,140 YOU WILL, FOR FURTHER INVEST M, 7674 04:40:21,140 --> 04:40:24,109 IF THERE'S A CLEAR GO/NO-GO FOR 7675 04:40:24,109 --> 04:40:25,944 PATIENT BENEFIT BASED UPON WELL 7676 04:40:25,944 --> 04:40:27,146 UNDERSTOOD BIOMARKERS, IT JUST 7677 04:40:27,146 --> 04:40:28,580 INVITES THAT MUCH MORE RESEARCH 7678 04:40:28,580 --> 04:40:32,184 IN THOSE SPACES. 7679 04:40:32,184 --> 04:40:34,019 THEN I THINK THE LAST TOPIC THAT 7680 04:40:34,019 --> 04:40:36,355 I RECALL WAS AROUND THE 7681 04:40:36,355 --> 04:40:38,957 IMPORTANCE OF BIOPSIES. 7682 04:40:38,957 --> 04:40:41,160 AND PARTICULARLY IN THE GENETIC 7683 04:40:41,160 --> 04:40:42,628 MEDICINE SPACE, IT'S EXTREMELY 7684 04:40:42,628 --> 04:40:45,431 IMPORTANT TO UNDERSTAND WHERE A 7685 04:40:45,431 --> 04:40:46,732 PATIENT STARTS OUT IN ORDER TO 7686 04:40:46,732 --> 04:40:48,300 BE ABLE TO ASSESS WHERE THEY'RE 7687 04:40:48,300 --> 04:40:50,035 GOING, AND I THINK THIS CAN BE 7688 04:40:50,035 --> 04:40:51,336 REALLY CHALLENGING AS WE'VE 7689 04:40:51,336 --> 04:40:55,808 HEARD IN THE CF SPACE AND IPF 7690 04:40:55,808 --> 04:40:57,276 AROUND THE LIMITED BIOPSIES THAT 7691 04:40:57,276 --> 04:40:58,444 ARE OUT THERE RIGHT NOW AND THE 7692 04:40:58,444 --> 04:40:59,978 CHALLENGES OF CONVINCING 7693 04:40:59,978 --> 04:41:01,714 PATIENTS WHAT THAT VALUE IS TO 7694 04:41:01,714 --> 04:41:04,016 GIVING IT NOT NECESSARILY END 7695 04:41:04,016 --> 04:41:07,619 STAGE DISEASE TYPE BIOPSIES, BUT 7696 04:41:07,619 --> 04:41:08,954 ALSO UNDERSTANDING THE 7697 04:41:08,954 --> 04:41:10,155 LONGITUDINAL DISEASE PROGRESSION 7698 04:41:10,155 --> 04:41:11,790 ASPECTS, AND JUST HOW IT'S SO 7699 04:41:11,790 --> 04:41:13,092 DIFFICULT TO UNDERSTAND 7700 04:41:13,092 --> 04:41:15,060 SOMETIMES WHAT THAT DISEASE 7701 04:41:15,060 --> 04:41:16,495 PROGRESSION LOOKS LIKE BECAUSE 7702 04:41:16,495 --> 04:41:18,130 MANY OF THOSE BIOPSIES JUST 7703 04:41:18,130 --> 04:41:19,531 SIMPLY AREN'T AVAILABLE FOR MANY 7704 04:41:19,531 --> 04:41:23,836 DIFFERENT DISEASES. 7705 04:41:23,836 --> 04:41:24,703 SO WE CONTINUE TO WORK THROUGH 7706 04:41:24,703 --> 04:41:27,206 AND TRY TO FIND INNOVATIVE OTHER 7707 04:41:27,206 --> 04:41:28,640 WAYS TO KIND OF GET SURROGATES 7708 04:41:28,640 --> 04:41:30,709 FOR THIS BUT ULTIMATELY THERE'S 7709 04:41:30,709 --> 04:41:31,477 NO SUBSTITUTE FOR HAVING THE 7710 04:41:31,477 --> 04:41:32,578 TRUE BIOPSIES. 7711 04:41:32,578 --> 04:41:33,645 JOHN, WAS THERE ANYTHING I 7712 04:41:33,645 --> 04:41:37,182 MISSED YOU WANTED TO TOUCH ON? 7713 04:41:37,182 --> 04:41:39,118 >> NO, I THINK YOU COVERED 7714 04:41:39,118 --> 04:41:40,519 EVERYTHING QUITE WELL. 7715 04:41:40,519 --> 04:41:41,854 >> GREAT. 7716 04:41:41,854 --> 04:41:46,125 THANK YOU. 7717 04:41:46,125 --> 04:41:47,359 >> SO I THINK FOR THE NEXT 7718 04:41:47,359 --> 04:41:48,660 STEPS, JUST THINKING ABOUT THE 7719 04:41:48,660 --> 04:41:51,263 THEMES ACROSS BOTH WORKSHOP 7720 04:41:51,263 --> 04:41:52,064 DAYS, AND SOME OF THE THINGS 7721 04:41:52,064 --> 04:41:53,532 THAT WE DISCUSSED IN THE 7722 04:41:53,532 --> 04:41:56,735 BREAKOUTS AS WELL AS ON THE 7723 04:41:56,735 --> 04:41:57,970 PANELS, I THINK THE CRITICAL 7724 04:41:57,970 --> 04:42:00,472 CHALLENGE HERE IS TO THINK ABOUT 7725 04:42:00,472 --> 04:42:02,775 WHAT THE COMMUNITY CAN AND 7726 04:42:02,775 --> 04:42:04,176 SHOULD DO, SORT OF 7727 04:42:04,176 --> 04:42:05,811 RECOMMENDATIONS AND IDEAS FOR 7728 04:42:05,811 --> 04:42:08,981 THE RESEARCH COMMUNITY VERSUS 7729 04:42:08,981 --> 04:42:12,584 WHAT WE CAN DO AT THE NIH TO 7730 04:42:12,584 --> 04:42:14,787 U.SUPPORT THOSE ACTIVITIES AS 7731 04:42:14,787 --> 04:42:15,721 WELL, LIKE WHAT SPECIFICALLY MAY 7732 04:42:15,721 --> 04:42:20,592 BE IN OUR DOMAIN AS 7733 04:42:20,592 --> 04:42:21,960 RECOMMENDATIONS FOR THINKING 7734 04:42:21,960 --> 04:42:23,862 ABOUT WAYS WE CAN LEVERAGE 7735 04:42:23,862 --> 04:42:25,731 EXISTING MECHANISMS, EXISTING 7736 04:42:25,731 --> 04:42:32,070 POLICIES AND THINGS LIKE THAT, 7737 04:42:32,070 --> 04:42:33,405 VERSES LIKE MAKING 7738 04:42:33,405 --> 04:42:34,039 RECOMMENDATIONS FOR THE 7739 04:42:34,039 --> 04:42:35,674 SCIENTIFIC AND CLINICAL RESEARCH 7740 04:42:35,674 --> 04:42:37,075 COMMUNITY, WHICH MAY BE HARD IN 7741 04:42:37,075 --> 04:42:41,680 THIS TOPIC AREA, BECAUSE WE HAVE 7742 04:42:41,680 --> 04:42:43,215 A LOT OF DIFFERENT STAKEHOLDERS, 7743 04:42:43,215 --> 04:42:43,549 RIGHT? 7744 04:42:43,549 --> 04:42:49,021 WE HAVE PATIENTS, ADVOCACY 7745 04:42:49,021 --> 04:42:50,989 GROUPS, INDUSTRY, WE HAVE 7746 04:42:50,989 --> 04:42:52,090 INVESTORS, REGULATORY BODIES AND 7747 04:42:52,090 --> 04:42:54,927 THEN WE HAVE THE ACTUAL 7748 04:42:54,927 --> 04:42:56,261 RESEARCHERS AND CLINICIANS 7749 04:42:56,261 --> 04:42:56,929 INVOLVED ACROSS THOSE. 7750 04:42:56,929 --> 04:43:00,399 SO I THINK THAT ACTION ITEMS 7751 04:43:00,399 --> 04:43:01,733 FROM THE WORKSHOP, WE DEFINITELY 7752 04:43:01,733 --> 04:43:03,468 WILL HAVE AN EXECUTIVE SUMMARY 7753 04:43:03,468 --> 04:43:04,236 WHICH WILL REFLECT A LOT OF 7754 04:43:04,236 --> 04:43:08,073 THIS, SO WE WANT TO MAKE SURE 7755 04:43:08,073 --> 04:43:09,608 THAT WE HAVE THOSE THEMES 7756 04:43:09,608 --> 04:43:10,709 ADDRESSED, AND CERTAINLY IF THE 7757 04:43:10,709 --> 04:43:13,545 GROUP WAS INTERESTED IN MAKING A 7758 04:43:13,545 --> 04:43:14,613 WHITE PAPER POSITIONAL 7759 04:43:14,613 --> 04:43:15,614 STATEMENT, WE COULD CERTAINLY 7760 04:43:15,614 --> 04:43:19,184 HELP SUPPORT THAT AS WELL. 7761 04:43:19,184 --> 04:43:20,552 AND THEN WE WANT TO CONTINUE TO 7762 04:43:20,552 --> 04:43:23,055 WORK IN THIS SPACE. 7763 04:43:23,055 --> 04:43:24,590 IT IS SORT OF NEW FOR US. 7764 04:43:24,590 --> 04:43:27,326 AGAIN, CATALYZE IS -- I'M NOT 7765 04:43:27,326 --> 04:43:29,928 THE LEAD OF CATALYZE, BUT WE HAD 7766 04:43:29,928 --> 04:43:32,231 THE LEAD OF CATALYZE HERE 7767 04:43:32,231 --> 04:43:33,565 YESTERDAY BUT THAT'S A PROGRAM 7768 04:43:33,565 --> 04:43:36,735 THAT'S A LITTLE BIT IN ITS 7769 04:43:36,735 --> 04:43:37,369 INFANCY. 7770 04:43:37,369 --> 04:43:40,239 AND THAT'S KIND OF WHY WE'RE 7771 04:43:40,239 --> 04:43:41,540 HERE, IS BECAUSE WE RECOGNIZE 7772 04:43:41,540 --> 04:43:42,741 THAT THOSE TYPES OF PROGRAMS ARE 7773 04:43:42,741 --> 04:43:43,709 NEEDED BUT THEN THERE ARE A LOT 7774 04:43:43,709 --> 04:43:45,811 OF OTHER AREAS THAT WE COULD BE 7775 04:43:45,811 --> 04:43:47,546 ADDRESSING BY WORKING TOGETHER 7776 04:43:47,546 --> 04:43:48,313 REALLY. 7777 04:43:48,313 --> 04:43:50,816 SO REALLY, KATELYN AND JEFF, IF 7778 04:43:50,816 --> 04:43:53,118 YOU HAVE IDEAS AND THINGS THAT 7779 04:43:53,118 --> 04:43:54,887 YOU THINK THAT WE SHOULD BE 7780 04:43:54,887 --> 04:43:58,490 CONVEYING TO THE COMMUNITY, WE'D 7781 04:43:58,490 --> 04:44:01,226 REALLY LIKE TO HEAR THAT. 7782 04:44:01,226 --> 04:44:03,195 >> I WAS GOING TO SAY, LADIES 7783 04:44:03,195 --> 04:44:06,698 FIRST, BUT -- NO, JUST -- I 7784 04:44:06,698 --> 04:44:08,433 THINK WE'VE TOUCHED ON A LOT OF 7785 04:44:08,433 --> 04:44:09,101 THOSE TOPICS. 7786 04:44:09,101 --> 04:44:12,070 I MEAN, I'M NOT AN EXPERT ON 7787 04:44:12,070 --> 04:44:13,038 DIFFERENT GOVERNMENT PROGRAMS 7788 04:44:13,038 --> 04:44:17,175 THAT ARE AVAILABLE ALREADY, BUT 7789 04:44:17,175 --> 04:44:18,710 IT JUST FEELS LIKE THESE NATURAL 7790 04:44:18,710 --> 04:44:20,479 HISTORY STUDIES, THE BIOMARKER 7791 04:44:20,479 --> 04:44:22,214 TYPE, FUNDING TO SUPPORT THOSE 7792 04:44:22,214 --> 04:44:25,717 ACTIVITIES ARE REALLY -- WILL 7793 04:44:25,717 --> 04:44:28,553 DIRECTLY BENEFIT SO MANY OF 7794 04:44:28,553 --> 04:44:29,988 THOSE INVESTED STAKEHOLDERS THAT 7795 04:44:29,988 --> 04:44:31,857 YOU'VE SPOKEN ABOUT TODAY, 7796 04:44:31,857 --> 04:44:33,492 MARRAH, AND THAT WE'VE ALL 7797 04:44:33,492 --> 04:44:34,293 DISCUSSED. 7798 04:44:34,293 --> 04:44:35,360 THOSE BENEFIT EVERYONE IN THE 7799 04:44:35,360 --> 04:44:39,298 PROCESS, AND I THINK ALSO WE'LL 7800 04:44:39,298 --> 04:44:41,133 DIRECTLY TRANSLATE TO PATIENT 7801 04:44:41,133 --> 04:44:43,769 BENEFIT OVERALL, SO I THINK 7802 04:44:43,769 --> 04:44:45,637 MECHANISMS AROUND THAT ARE A 7803 04:44:45,637 --> 04:44:46,605 GREAT PLACE TO START 7804 04:44:46,605 --> 04:44:49,574 POTENTIALLY. 7805 04:44:49,574 --> 04:44:54,947 >> CONTINUING THE CONNECTION OF 7806 04:44:54,947 --> 04:44:56,348 THE DIFFERENT SILOS OF RESEARCH 7807 04:44:56,348 --> 04:44:57,549 TOGETHER TO HAVE THESE 7808 04:44:57,549 --> 04:44:58,483 CONVERSATIONS, I THINK SERVES AS 7809 04:44:58,483 --> 04:45:02,254 A REALLY IMPORTANT SPARK TO MANY 7810 04:45:02,254 --> 04:45:06,625 OF THESE EFFORTS. 7811 04:45:06,625 --> 04:45:08,193 SO -- AND YOU KIND OF SEE PEOPLE 7812 04:45:08,193 --> 04:45:09,494 LIGHT UP IN A WAY THAT THEY 7813 04:45:09,494 --> 04:45:10,996 DON'T IN THEIR REGULAR JOB WHEN 7814 04:45:10,996 --> 04:45:15,167 THEY'RE ABLE TO KIND OF 7815 04:45:15,167 --> 04:45:15,934 CROSS-POLLINATE WITH THESE OTHER 7816 04:45:15,934 --> 04:45:17,369 DISEASE SPACES AND THAT 7817 04:45:17,369 --> 04:45:18,537 INTERACTION ONLY MAKES US 7818 04:45:18,537 --> 04:45:21,173 STRONGER, SO I THINK ONGOING 7819 04:45:21,173 --> 04:45:23,375 EFFORTS TO SUPPORT THAT, I THINK 7820 04:45:23,375 --> 04:45:26,545 A LITTLE BIT OF BRAINSTORMING 7821 04:45:26,545 --> 04:45:27,312 DISCUSSION ABOUT WHAT COULD 7822 04:45:27,312 --> 04:45:28,714 HAPPEN IN CONJUNCTION WITH ATS 7823 04:45:28,714 --> 04:45:30,482 AND ARE THERE WAYS TO FOCUS IN 7824 04:45:30,482 --> 04:45:33,652 ON SOME OF THIS KIND OF MORE 7825 04:45:33,652 --> 04:45:34,987 EARLY RESEARCH TO TRY TO CONNECT 7826 04:45:34,987 --> 04:45:36,288 PEOPLE IN THAT SPACE. 7827 04:45:36,288 --> 04:45:37,723 AND I THINK FROM THE COMMUNITY, 7828 04:45:37,723 --> 04:45:39,324 IT SOUNDS LIKE WE'VE GOT SOME 7829 04:45:39,324 --> 04:45:41,426 GOOD RECOMMENDATIONS FOR ONGOING 7830 04:45:41,426 --> 04:45:42,861 WORK PARTICULARLY AROUND STUDY 7831 04:45:42,861 --> 04:45:47,332 END POINTS AND HOW WE HELP THE 7832 04:45:47,332 --> 04:45:50,302 FDA WITH THE INFORMATION THEY 7833 04:45:50,302 --> 04:45:53,005 NEED TO MAKE DRUG APPROVAL 7834 04:45:53,005 --> 04:45:54,539 EASIER FOR US. 7835 04:45:54,539 --> 04:45:57,843 SO I FEEL LIKE A LOT OF THAT 7836 04:45:57,843 --> 04:45:59,378 CONVERSATION IS IMPORTANT AND 7837 04:45:59,378 --> 04:46:04,182 WILL CERTAINLY BE GOOD TO PUT IN 7838 04:46:04,182 --> 04:46:06,918 WRITING. 7839 04:46:06,918 --> 04:46:11,289 YEAH. 7840 04:46:11,289 --> 04:46:12,357 I CAN'T HAVE THE FINAL WORD. 7841 04:46:12,357 --> 04:46:14,226 >> I'M GOING TO ASK JOHN IF HE 7842 04:46:14,226 --> 04:46:16,094 HAD ANY OVERARCHING THEMES OR 7843 04:46:16,094 --> 04:46:17,729 THINGS THAT HE WANTED TO TOUCH 7844 04:46:17,729 --> 04:46:20,499 ON AS WELL. 7845 04:46:20,499 --> 04:46:22,534 >> NO, NOT PARTICULARLY. 7846 04:46:22,534 --> 04:46:24,736 I THINK THIS HAS BEEN A GREAT 7847 04:46:24,736 --> 04:46:26,071 DISCUSSION, I THINK IT'S BEEN A 7848 04:46:26,071 --> 04:46:28,673 REALLY GREAT TWO DAYS, LOTS OF 7849 04:46:28,673 --> 04:46:29,341 FANTASTIC TALKS AND 7850 04:46:29,341 --> 04:46:31,309 PRESENTATIONS. 7851 04:46:31,309 --> 04:46:32,744 AS MARRAH SAID, I THINK THERE'S 7852 04:46:32,744 --> 04:46:36,114 A LOT OF INFORMATION THAT WE'LL 7853 04:46:36,114 --> 04:46:38,417 TAKE AND WORK TOGETHER TO PUT 7854 04:46:38,417 --> 04:46:40,385 TOGETHER THE EXECUTIVE SUMMARY 7855 04:46:40,385 --> 04:46:41,787 AND DISSEMINATE FINDINGS FROM 7856 04:46:41,787 --> 04:46:42,888 THIS TO THE COMMUNITY. 7857 04:46:42,888 --> 04:46:46,858 SO BACK TO YOU. 7858 04:46:46,858 --> 04:46:50,762 >> I THINK THAT THIS DOES POSE A 7859 04:46:50,762 --> 04:46:52,964 CHALLENGE AS KATELYN YOU NOTED, 7860 04:46:52,964 --> 04:46:53,932 LIKE THE CONVERSATION CAN'T 7861 04:46:53,932 --> 04:46:56,568 REALLY STOP WITH TODAY. 7862 04:46:56,568 --> 04:47:00,505 AND WHILE WE CAN CONTINUE THE 7863 04:47:00,505 --> 04:47:03,241 MOMENTUM, MAYBE SMALL PIECES OF 7864 04:47:03,241 --> 04:47:04,776 WORKING TOGETHER WITH THE 7865 04:47:04,776 --> 04:47:05,877 ADVOCACY GROUPS THAT HAVE 7866 04:47:05,877 --> 04:47:08,046 PLATFORMS FOR CONNECTIONS TO 7867 04:47:08,046 --> 04:47:12,984 INVESTIGATORS AND PATIENTS AND 7868 04:47:12,984 --> 04:47:16,021 HOW THEY INCENTIVIZE INVESTMENT 7869 04:47:16,021 --> 04:47:17,489 FROM THE COMMUNITY WHICH THE 7870 04:47:17,489 --> 04:47:20,926 RESOURCES THEY HAVE, AND SO HOW 7871 04:47:20,926 --> 04:47:22,828 WE COULD HELP MAKE THOSE 7872 04:47:22,828 --> 04:47:26,098 CONNECTIONS AT SMALLER LEVELS, 7873 04:47:26,098 --> 04:47:27,432 WHETHER THAT'S HAVING A LITTLE 7874 04:47:27,432 --> 04:47:30,602 MORE FOCUS ON RARE DISEASES AND 7875 04:47:30,602 --> 04:47:32,404 UNMET NEEDS AT THE INNOVATION 7876 04:47:32,404 --> 04:47:33,972 SUMMIT AS YOU'RE THINKING ABOUT 7877 04:47:33,972 --> 04:47:37,042 ATS AND I KNOW THAT MIKE HAD 7878 04:47:37,042 --> 04:47:39,344 MENTIONED YESTERDAY THINKING 7879 04:47:39,344 --> 04:47:40,712 ABOUT OTHER CONFERENCE AND 7880 04:47:40,712 --> 04:47:42,948 PLATFORM LEVELS FOR THIS TO 7881 04:47:42,948 --> 04:47:44,382 HAPPEN IN THE LUNG COMMUNITY. 7882 04:47:44,382 --> 04:47:48,620 AND YOU KNOW, THINKING THE ABOE 7883 04:47:48,620 --> 04:47:49,821 DIFFERENCES BETWEEN INSTITUTES 7884 04:47:49,821 --> 04:47:51,156 AND CENTERS, WE ALL SORT OF SET 7885 04:47:51,156 --> 04:47:52,791 OURSELVES AROUND A MISSION THAT 7886 04:47:52,791 --> 04:47:54,860 COMPLEMENTS THE NIH'S MISSION AS 7887 04:47:54,860 --> 04:47:57,362 ROB HAD MENTIONED, WE WANT TO 7888 04:47:57,362 --> 04:47:59,564 TURN DISCOVERIES INTO HEALTH, 7889 04:47:59,564 --> 04:48:01,199 AND THIS CONVERSATION IS REALLY 7890 04:48:01,199 --> 04:48:03,401 SAYING THERE ARE BARRIERS TO US 7891 04:48:03,401 --> 04:48:04,870 DOING THAT, SO WE NEED TO FIGURE 7892 04:48:04,870 --> 04:48:07,472 OUT WHAT THOSE BARRIERS ARE AS A 7893 04:48:07,472 --> 04:48:09,307 COMMUNITY AND LEVERAGE WHAT WE 7894 04:48:09,307 --> 04:48:10,742 HAVE AND THEN ALSO FIGURE OUT 7895 04:48:10,742 --> 04:48:13,578 WAYS TO BE MORE CREATIVE 7896 04:48:13,578 --> 04:48:13,912 TOGETHER. 7897 04:48:13,912 --> 04:48:15,814 AND THIS IS I THINK OF CRITICAL 7898 04:48:15,814 --> 04:48:18,083 IMPORTANCE TO THE RARE DISEASE 7899 04:48:18,083 --> 04:48:20,051 COMMUNITY AND UNMET NEEDS WHY WE 7900 04:48:20,051 --> 04:48:22,220 HAD THAT FOCUS OF THIS 7901 04:48:22,220 --> 04:48:25,857 PARTICULAR WORKSHOP BECAUSE 7902 04:48:25,857 --> 04:48:29,761 THERE ARE A LOT OF DISEASES WITH 7903 04:48:29,761 --> 04:48:30,762 EITHER SOMEWHAT DISEASE 7904 04:48:30,762 --> 04:48:33,498 MODIFYING THERAPIES OR NO 7905 04:48:33,498 --> 04:48:34,599 THERAPIES, AND ESSENTIALLY MOST 7906 04:48:34,599 --> 04:48:36,768 OF THEM HAVE NO CURES. 7907 04:48:36,768 --> 04:48:39,304 AND SO THAT'S LIKE REALLY THE 7908 04:48:39,304 --> 04:48:41,773 HEALTH EQUITY ISSUE FOR THOSE 7909 04:48:41,773 --> 04:48:43,842 INDIVIDUALS WITH HIGH MORBIDITY 7910 04:48:43,842 --> 04:48:45,410 AND MORTALITY IS SOMETHING 7911 04:48:45,410 --> 04:48:47,979 THAT'S A CRITICAL IMPORTANCE, SO 7912 04:48:47,979 --> 04:48:49,347 REALLY JUST FIGURING OUT WAYS TO 7913 04:48:49,347 --> 04:48:54,286 BREAK THOSE BARRIERS, AND NIH IS 7914 04:48:54,286 --> 04:48:55,387 JUST ONE PIECE AND SO HOW WE CAN 7915 04:48:55,387 --> 04:48:57,022 BE MORE CREATIVE WITH OUR 7916 04:48:57,022 --> 04:48:59,658 FEDERAL PARTNERS, I THINK IS 7917 04:48:59,658 --> 04:49:01,393 SOMETHING THAT IS GOING TO BE 7918 04:49:01,393 --> 04:49:03,028 SOMETHING WE SHOULD CONSIDER AND 7919 04:49:03,028 --> 04:49:05,430 CONTINUE TO WORK ON, ESPECIALLY 7920 04:49:05,430 --> 04:49:07,299 IN THE RARE DISEASE SPACE. 7921 04:49:07,299 --> 04:49:10,368 SO A LOT OF IDEAS CAME OUT 7922 04:49:10,368 --> 04:49:12,003 AROUND THOSE THEMES AND REALLY 7923 04:49:12,003 --> 04:49:13,004 DO APPRECIATE ALL THE SPEAKERS 7924 04:49:13,004 --> 04:49:15,707 AND THE TIME THAT THEY'VE GIVEN 7925 04:49:15,707 --> 04:49:18,476 TODAY, AND ALSO FOR YOU ALL, 7926 04:49:18,476 --> 04:49:20,111 HELPING PLAN THIS WONDERFUL 7927 04:49:20,111 --> 04:49:21,012 WORKSHOP AND DEDICATING YOUR 7928 04:49:21,012 --> 04:49:22,614 TIME AND EFFORT TO ALL OF IT. 7929 04:49:22,614 --> 04:49:24,816 AND IT REALLY MUCH APPRECIATED. 7930 04:49:24,816 --> 04:49:26,451 SO WITH THAT, I THINK WE CAN 7931 04:49:26,451 --> 04:49:29,621 CLOSE OUT, UNLESS YOU HAVE 7932 04:49:29,621 --> 04:49:30,622 ANYTHING ELSE YOU WANT TO SAY 7933 04:49:30,622 --> 04:49:33,592 BEFORE WE DO? 7934 04:49:33,592 --> 04:49:35,227 >> JUST THANK YOU, MARA AND 7935 04:49:35,227 --> 04:49:39,164 JOHN, FOR HOSTING, YOU DO AN 7936 04:49:39,164 --> 04:49:40,899 AMAZING JOB HOSTING THIS. 7937 04:49:40,899 --> 04:49:42,100 THRILLED TO HELP, AND I THINK 7938 04:49:42,100 --> 04:49:42,968 THERE WAS A LOT OF PROGRESS 7939 04:49:42,968 --> 04:49:43,969 DURING THIS MEETING, SO THANK 7940 04:49:43,969 --> 04:49:44,135 YOU. 7941 04:49:44,135 --> 04:49:46,438 >> YES, THANK YOU, EVERYBODY. 7942 04:49:46,438 --> 04:49:48,540 >> THANK YOU, GUYS. 7943 04:49:48,540 --> 04:49:49,374 >> THANK YOU. 7944 04:49:49,374 --> 04:49:50,075 >> BYE. 7945 04:49:50,075 --> 04:49:50,842 >> BYE. 7946 04:49:50,842 --> 04:50:01,086 >> THANK YOU.