1 00:00:06,040 --> 00:00:09,920 >> LET'S GET STARTED. 2 00:00:09,920 --> 00:00:13,480 FIRST OF ALL I WOULD SAY MY 3 00:00:13,480 --> 00:00:15,240 BRAIN HAS BEEN GOING INTO 4 00:00:15,240 --> 00:00:17,440 HYPERSPEED, I DON'T KNOW IF THE 5 00:00:17,440 --> 00:00:18,600 CONVERSATIONS YESTERDAY, 6 00:00:18,600 --> 00:00:20,200 THINKING ABOUT THE LOT OF THE 7 00:00:20,200 --> 00:00:22,400 COMMENTS MADE BOTH IN CLOSE AND 8 00:00:22,400 --> 00:00:23,720 OPEN SESSION SO THANK YOU FOR A 9 00:00:23,720 --> 00:00:24,560 WONDERFUL DISCUSSION YESTERDAY 10 00:00:24,560 --> 00:00:27,160 AND I LOOK FORWARD TO 11 00:00:27,160 --> 00:00:28,160 DISCUSSIONS TODAY. 12 00:00:28,160 --> 00:00:32,040 I KNOW IT WILL BE EQUALLY 13 00:00:32,040 --> 00:00:34,120 INTERESTING. 14 00:00:34,120 --> 00:00:36,640 I THINK BEFORE WE GET STARTED, 15 00:00:36,640 --> 00:00:37,880 WE PROBABLY SHOULD QUICK ROLL 16 00:00:37,880 --> 00:00:40,880 CALL TO MAKE SURE WE HAVE 17 00:00:40,880 --> 00:00:42,360 EVERYBODY HERE IN TOTAL CAPACITY 18 00:00:42,360 --> 00:00:44,280 AND THEN WE WILL RUN THROUGH THE 19 00:00:44,280 --> 00:00:47,320 AGENDA WITH YOU AND GET STARTED. 20 00:00:47,320 --> 00:00:49,680 ANNA, I WILL TURN IT TO YOU FOR 21 00:00:49,680 --> 00:00:51,360 QUICK ROLL CALL BEFORE WE START. 22 00:00:51,360 --> 00:00:54,560 >> THANK YOU, JONI. 23 00:00:54,560 --> 00:00:55,040 PAUL HARRIS. 24 00:00:55,040 --> 00:00:56,920 >> HERE. 25 00:00:56,920 --> 00:01:02,160 >> KRISTINA HARTMAN. 26 00:01:02,160 --> 00:01:04,720 >> HERE. 27 00:01:04,720 --> 00:01:06,200 >> TED HOLMAN. 28 00:01:06,200 --> 00:01:07,000 >> HERE. 29 00:01:07,000 --> 00:01:12,160 >> REBECCA JACKSON 30 00:01:12,160 --> 00:01:13,040 >> HERE. 31 00:01:13,040 --> 00:01:15,120 >> ANNIE KENNEDY. 32 00:01:15,120 --> 00:01:18,200 >> HERE. 33 00:01:18,200 --> 00:01:20,960 >> MATTHIAS KRETZLER. 34 00:01:20,960 --> 00:01:25,000 >> YEP. 35 00:01:25,000 --> 00:01:28,360 KELLY MCVEARRY, HERE. 36 00:01:28,360 --> 00:01:29,560 >> KEITH MULLER. 37 00:01:29,560 --> 00:01:31,320 >> HERE. 38 00:01:31,320 --> 00:01:34,080 >> RAJESH RANGANATHAN. 39 00:01:34,080 --> 00:01:35,560 >> HERE. 40 00:01:35,560 --> 00:01:36,400 PAULA SHIREMAN. 41 00:01:36,400 --> 00:01:40,400 >> HERE. 42 00:01:40,400 --> 00:01:41,080 MARSHALL SUMMAR. 43 00:01:41,080 --> 00:01:44,560 >> HERE. 44 00:01:44,560 --> 00:01:46,800 RA JESH 45 00:01:46,800 --> 00:01:55,480 >> THANK YOU, THE NEXT SLIDE. 46 00:01:55,480 --> 00:01:57,880 >> YOU ARE IN THE PANEL SO YOU 47 00:01:57,880 --> 00:02:00,000 ARE FREE TO SPEAK HOWEVER TO 48 00:02:00,000 --> 00:02:01,680 MAINTAIN A SMOOTH PRESENTATION 49 00:02:01,680 --> 00:02:02,920 USE YOUR RAISE HAND FEATURE AND 50 00:02:02,920 --> 00:02:04,960 WAIT UNTIL YOU ARE WALLED UPON. 51 00:02:04,960 --> 00:02:06,160 PLEASE ALLOW THE SPEAKERS TO 52 00:02:06,160 --> 00:02:08,160 MOVE THROUGH THEIR PRESENTATIONS 53 00:02:08,160 --> 00:02:11,360 NOTING YOUR COMMENTS WHEN CALLED 54 00:02:11,360 --> 00:02:11,880 UPON. 55 00:02:11,880 --> 00:02:14,640 WE ASK YOU USE CHAT JUDICIOUSLY 56 00:02:14,640 --> 00:02:16,360 BECAUSE THE COMMENTS ARE OF 57 00:02:16,360 --> 00:02:17,280 COURSE INTEGRATED INTO THE 58 00:02:17,280 --> 00:02:18,440 MINUTES. 59 00:02:18,440 --> 00:02:19,440 SO IT BECOMES PART OF THE 60 00:02:19,440 --> 00:02:24,760 RECORD. 61 00:02:24,760 --> 00:02:27,560 WE ARE ALSO LEUSING THE 62 00:02:27,560 --> 00:02:29,760 VIDEOCAST FEATURE, SO MEMBERSES 63 00:02:29,760 --> 00:02:31,520 IN THE AUDIENCE USE THE EMAIL 64 00:02:31,520 --> 00:02:33,120 FUNCTION ON THE NIH VIDEOCAST 65 00:02:33,120 --> 00:02:35,440 SITE USING THE BUTTON AVAILABLE 66 00:02:35,440 --> 00:02:37,440 OR SENDING EMAIL TO NCATS 67 00:02:37,440 --> 00:02:46,800 COUNCIL INPUT MAILBOX. 68 00:02:46,800 --> 00:02:48,480 FUTURE MEETING DATES. 69 00:02:48,480 --> 00:02:50,080 IN 2022 WE ARE ANTICIPATING A 70 00:02:50,080 --> 00:02:52,360 FACE TO FACE NEEDING MAY 19 AND 71 00:02:52,360 --> 00:02:54,920 SEPTEMBER 22ND. 72 00:02:54,920 --> 00:02:56,160 IN 2023 WE ARE PLANNING A 73 00:02:56,160 --> 00:02:58,960 VIRTUAL MEETING ON JANUARY 26 74 00:02:58,960 --> 00:03:01,760 AND 27, ON MAY 25 AND SEPTEMBER 75 00:03:01,760 --> 00:03:03,960 28. 76 00:03:03,960 --> 00:03:06,560 THE MAY MEETING IN 2022, THE 77 00:03:06,560 --> 00:03:07,640 NEXT MEETING CURRENTLY IS 78 00:03:07,640 --> 00:03:09,920 PLANNED FOR FACE TO FACE MEETING 79 00:03:09,920 --> 00:03:10,960 BUT CIRCUMSTANCES MAY DICTATE 80 00:03:10,960 --> 00:03:14,160 THAT THAT MEETINGS ALSO -- SO 81 00:03:14,160 --> 00:03:15,400 STAY TUNED WHETHER OR NOT WE ARE 82 00:03:15,400 --> 00:03:19,680 FACE TO FACE OR VIRTUAL IN MAY. 83 00:03:19,680 --> 00:03:20,760 MAY I HAVE THE NEXT SLIDE 84 00:03:20,760 --> 00:03:22,520 PLEASE. 85 00:03:22,520 --> 00:03:24,160 AS YOU CAN SEE TODAY WE WILL 86 00:03:24,160 --> 00:03:25,560 HAVE A PROGRAM UPDATE FROM THE 87 00:03:25,560 --> 00:03:27,360 OFFICE OF RARE DISEASE RESEARCH. 88 00:03:27,360 --> 00:03:29,280 THEN WE WILL HAVE THE CLEARANCE 89 00:03:29,280 --> 00:03:30,080 OF TWO CONCEPTS. 90 00:03:30,080 --> 00:03:33,320 WE WILL HAVE THE NCATS TRIENNIAL 91 00:03:33,320 --> 00:03:34,320 INCLUSION REPORT, THE PUBLIC 92 00:03:34,320 --> 00:03:35,880 HEARING OF ORGANIZATIONAL 93 00:03:35,880 --> 00:03:37,640 CHANGES AT NCATS AND THEN WE 94 00:03:37,640 --> 00:03:41,000 WILL ADJOURN. 95 00:03:41,000 --> 00:03:56,960 WITH THAT, NEXT SLIDE, WITH THAT 96 00:03:56,960 --> 00:03:59,560 WE'LL TURN IT OVER TO JONI NOW 97 00:03:59,560 --> 00:04:00,760 TO INTRODUCE OUR PROGRAM 98 00:04:00,760 --> 00:04:01,880 UPDATES. 99 00:04:01,880 --> 00:04:03,800 >> THANK YOU, ANNA. 100 00:04:03,800 --> 00:04:06,160 I THINK THIS PROGRAM NEEDS TO 101 00:04:06,160 --> 00:04:07,000 INTRODUCTION. 102 00:04:07,000 --> 00:04:08,240 I GAVE IT YESTERDAY BUT VERY 103 00:04:08,240 --> 00:04:11,520 EXCITED TO HAVE ANNE PARISER 104 00:04:11,520 --> 00:04:17,960 WITH HER LAST UPDATE FOR THIS 105 00:04:17,960 --> 00:04:19,960 COUNCIL AS DIRECTOR OF OFFICE OF 106 00:04:19,960 --> 00:04:21,760 RARE DISEASE RESEARCH AND ALSO 107 00:04:21,760 --> 00:04:23,760 TO GIVE A WARM HAND OFF TO PJ 108 00:04:23,760 --> 00:04:25,960 BROOKS WHO WILL STEP IN AS THE 109 00:04:25,960 --> 00:04:27,320 ACTING DIRECTOR AND HE CURRENTLY 110 00:04:27,320 --> 00:04:28,640 SERVINGS AS DEPUTY DIRECTOR SO 111 00:04:28,640 --> 00:04:30,560 THEY WILL DO A LITTLE TAG TEAM 112 00:04:30,560 --> 00:04:32,440 HERE AND IT IS A PRETTY RICH 113 00:04:32,440 --> 00:04:33,920 PRESENTATION SO I WILL -- 114 00:04:33,920 --> 00:04:34,880 WITHOUT FURTHER ADIEU I WILL 115 00:04:34,880 --> 00:04:37,360 TURN IT OVER TO YOU, ANNE. 116 00:04:37,360 --> 00:04:39,120 GO AHEAD. 117 00:04:39,120 --> 00:04:41,360 >> GOOD AFTERNOON, EVERYONE. 118 00:04:41,360 --> 00:04:44,000 I AM NOT ACTUALLY PJ BROOKS, I'M 119 00:04:44,000 --> 00:04:46,360 ANNE PARISER, DIRECTOR OF OFFICE 120 00:04:46,360 --> 00:04:47,960 OF RARE DISEASE RESEARCH AND PJ 121 00:04:47,960 --> 00:04:50,480 IS SITTING RIGHT HERE IN THE 122 00:04:50,480 --> 00:04:54,320 SAME ROOM, AS JONI SAID WE WILL 123 00:04:54,320 --> 00:04:56,360 BE DOING COMBINED PRESENTATION. 124 00:04:56,360 --> 00:04:59,760 SO I'M GOING TO TRY TO SHARE 125 00:04:59,760 --> 00:05:01,120 SCREEN HERE. 126 00:05:01,120 --> 00:05:06,960 SO WE CAN CONTROL. 127 00:05:06,960 --> 00:05:09,560 >> IT IS RARE TO HAVE TWO PEOPLE 128 00:05:09,560 --> 00:05:11,400 SITTING IN THE SAME ROOM WITHOUT 129 00:05:11,400 --> 00:05:11,960 MASKS. 130 00:05:11,960 --> 00:05:12,560 >> YES, IT IS. 131 00:05:12,560 --> 00:05:12,960 ALL RIGHT. 132 00:05:12,960 --> 00:05:16,600 SO CAN EVERYBODY SEE THE INTRO 133 00:05:16,600 --> 00:05:17,800 SLIDE? 134 00:05:17,800 --> 00:05:19,360 IT SHOULD SAY PROGRAM UPDATE 135 00:05:19,360 --> 00:05:21,360 OFFICE OF RARE DISEASE RESEARCH. 136 00:05:21,360 --> 00:05:22,560 >> ALL GOOD. 137 00:05:22,560 --> 00:05:24,280 >> YES. 138 00:05:24,280 --> 00:05:26,960 >> PJ YOU HAVE FULL CONTROL 139 00:05:26,960 --> 00:05:27,360 ANNE. 140 00:05:27,360 --> 00:05:28,760 >> THANK YOU. 141 00:05:28,760 --> 00:05:32,320 SO JUST BY WAY OF INTRO, I JUST 142 00:05:32,320 --> 00:05:35,160 TALK ABOUT OUR MISSION. 143 00:05:35,160 --> 00:05:37,920 IT IS ONE HAND VERY SIMPLE BUT 144 00:05:37,920 --> 00:05:38,480 VERY BROAD. 145 00:05:38,480 --> 00:05:40,200 WE SEEK TO ADVANCE RARE DISEASE 146 00:05:40,200 --> 00:05:41,400 RESEARCH TO BENEFIT OUR 147 00:05:41,400 --> 00:05:41,960 PATIENTS. 148 00:05:41,960 --> 00:05:44,160 AND WE DO THIS BY TRYING TO 149 00:05:44,160 --> 00:05:46,240 IMPROVE THE RESEARCH PROCESS FOR 150 00:05:46,240 --> 00:05:49,440 RARE DISEASES NOT FOR ANY ONE 151 00:05:49,440 --> 00:05:51,040 PARTICULAR RARE DISEASE BUT TO 152 00:05:51,040 --> 00:05:53,040 TRY TO IMPACT THE ENTIRE RARE 153 00:05:53,040 --> 00:05:55,760 DISEASE ECOSYSTEM. 154 00:05:55,760 --> 00:05:57,440 THIS SELECTION OF THIS PICTURE 155 00:05:57,440 --> 00:05:59,400 IS WE THINK CURRENTLY WE ARE 156 00:05:59,400 --> 00:06:01,400 ONLY LIMITED BY AS FAR AS WE ARE 157 00:06:01,400 --> 00:06:04,560 WILLING TO SEE. 158 00:06:04,560 --> 00:06:06,600 >> ANNE, SORRY TO INTERRUPT. 159 00:06:06,600 --> 00:06:08,000 ARE YOU STILL ON THE TITLE 160 00:06:08,000 --> 00:06:08,400 SLIDE? 161 00:06:08,400 --> 00:06:10,000 >> IS IT STUCK? 162 00:06:10,000 --> 00:06:13,840 >> IT'S STUCK. 163 00:06:13,840 --> 00:06:16,280 >> HIT THE ARROW TO GO O THE 164 00:06:16,280 --> 00:06:24,560 NEXT SLIDE. 165 00:06:24,560 --> 00:06:27,160 >> CAN YOU SEE IT NOW? 166 00:06:27,160 --> 00:06:28,760 WE ARE STILL ON THE TITLE. 167 00:06:28,760 --> 00:06:30,680 >> STILL TITLE SLIDE. 168 00:06:30,680 --> 00:06:33,200 >> MIKE IS ON HIS WAY TO HELP 169 00:06:33,200 --> 00:06:34,840 YOU GUYS. 170 00:06:34,840 --> 00:06:39,160 >> I'LL TRY SHARE SCREEN AGAIN. 171 00:06:39,160 --> 00:06:40,080 >> ANNE. 172 00:06:40,080 --> 00:06:41,760 >> YES. 173 00:06:41,760 --> 00:06:47,160 >> WAIT UNTIL MI MIKE ARRIVES. 174 00:06:47,160 --> 00:06:53,920 >> HE'LL BE HERE MOMENTARILY. 175 00:06:53,920 --> 00:06:56,240 >> I DON'T KNOW WHO MIKE IS BUT 176 00:06:56,240 --> 00:06:58,000 HE'S GETTING HIS STEPS IN TODAY 177 00:06:58,000 --> 00:07:03,360 FROM COUNCIL TO COUNCIL. 178 00:07:03,360 --> 00:07:07,200 >> MOST OF NCATS IS 9TH AND 10TH 179 00:07:07,200 --> 00:07:09,520 FLOOR AND OFFICE OF RARE DISEASE 180 00:07:09,520 --> 00:07:10,560 RESEARCH IS ON THE SECOND FLOOR 181 00:07:10,560 --> 00:07:16,560 SO E H' HE'S DEFINITELY GETTING STEPS 182 00:07:16,560 --> 00:07:17,040 IN. 183 00:07:17,040 --> 00:07:21,960 THERE MIGHT BE STAIRS. 184 00:07:21,960 --> 00:07:24,520 >> IS IT OKAY I MENTION THE 185 00:07:24,520 --> 00:07:28,160 MEETING WE HAD WITH RKCR 186 00:07:28,160 --> 00:07:29,080 LEADERSHIP WHILE WAITING? 187 00:07:29,080 --> 00:07:31,360 >> SURE. 188 00:07:31,360 --> 00:07:34,920 MIKE IS HERE. 189 00:07:34,920 --> 00:07:36,600 >> IN THAT CASE I'LL SAVE IT. 190 00:07:36,600 --> 00:07:38,000 >> GO AHEAD MARSHALL. 191 00:07:38,000 --> 00:07:38,960 >> SURE. 192 00:07:38,960 --> 00:07:44,000 SO I'M ONE OF THE ADVISORY 193 00:07:44,000 --> 00:07:44,960 COUNSELORS FOR THE PLAQUE 194 00:07:44,960 --> 00:07:46,640 WOMEN'S HEALTH IMPEARTIVE RARE 195 00:07:46,640 --> 00:07:48,160 DISEASE INITIATIVE. 196 00:07:48,160 --> 00:07:50,600 ONE THING WE TRYING TO DO IS GET 197 00:07:50,600 --> 00:07:52,520 BASELINE OF DATA AROUND 198 00:07:52,520 --> 00:07:53,560 PARTICIPATION OF 199 00:07:53,560 --> 00:07:55,000 UNDER-REPRESENTED MINORITIES AND 200 00:07:55,000 --> 00:07:56,560 DIFFERENT ETHNIC GROUPS AND RARE 201 00:07:56,560 --> 00:07:57,080 DISEASE RESEARCH. 202 00:07:57,080 --> 00:07:59,040 WE HAD A LITTLE BIT OF 203 00:07:59,040 --> 00:08:00,160 PRELIMINARY DATA SUGGESTING IT 204 00:08:00,160 --> 00:08:02,360 WAS LOW BUT BEFORE WE STARTED 205 00:08:02,360 --> 00:08:04,480 DOING ADVOCACY FOR MAJOR POLICY 206 00:08:04,480 --> 00:08:06,480 OR EFFORTS WE WANTED TO GET SOME 207 00:08:06,480 --> 00:08:08,320 REAL DATA AND SO WE CAME TO WHAT 208 00:08:08,320 --> 00:08:12,160 I CONSIDER THE LARGEST BEST RARE 209 00:08:12,160 --> 00:08:13,680 DISEASE LONG TERM STUDIES GOING 210 00:08:13,680 --> 00:08:15,640 ON SINCE 2004 AND WHAT WE ARE 211 00:08:15,640 --> 00:08:18,960 DOING IS LOOKING TO CONSOLIDATE 212 00:08:18,960 --> 00:08:21,400 DEIDENTIFY DATA FROM THE ANNUAL 213 00:08:21,400 --> 00:08:22,960 REPORTS ON PATIENT ENROLLMENT 214 00:08:22,960 --> 00:08:25,040 FROM THAT. 215 00:08:25,040 --> 00:08:27,040 ACROSS THE PATIENTS. 216 00:08:27,040 --> 00:08:28,440 REPRESENTS OVER 50,000 PATIENTS. 217 00:08:28,440 --> 00:08:30,640 IN MANY WAYS DEALING WITH 218 00:08:30,640 --> 00:08:33,280 LARGEST RARE DISEASE REPORTS 219 00:08:33,280 --> 00:08:35,480 FROM THAT MET WITH LEADERSHIP 220 00:08:35,480 --> 00:08:37,240 FROM PIs AND THEY ARE 221 00:08:37,240 --> 00:08:40,240 RECEPTIVE TO THAT, WILLING TO DO 222 00:08:40,240 --> 00:08:41,600 MORE BUT FIRST SEE WHAT THE BASE 223 00:08:41,600 --> 00:08:43,400 IS BEFORE WE GO AND DO SOME 224 00:08:43,400 --> 00:08:44,720 OTHER THINGS. 225 00:08:44,720 --> 00:08:46,240 SEE WHAT THE REAL ISSUE IS. 226 00:08:46,240 --> 00:08:48,160 >> THAT SOUNDS GREAT MARSHALL. 227 00:08:48,160 --> 00:08:49,920 YOU MENTION IN YOUR REPORT DOES 228 00:08:49,920 --> 00:08:50,920 THAT MEAN SOMETHING WILL COME 229 00:08:50,920 --> 00:08:54,200 OUT OF THIS? 230 00:08:54,200 --> 00:08:58,120 >> IT'S IN CONJUNCTION WITH RDRC 231 00:08:58,120 --> 00:08:59,760 MAKING SURE THAT'S WITH ALL 232 00:08:59,760 --> 00:09:01,280 RULES REGULATIONS AND THINGS 233 00:09:01,280 --> 00:09:02,320 LIKE THAT. 234 00:09:02,320 --> 00:09:04,480 THOUGH ANNUAL REPORTS ARE PUBLIC 235 00:09:04,480 --> 00:09:05,960 DOMAIN WE DIDN'T WANT TO GO THAT 236 00:09:05,960 --> 00:09:06,360 ROUTE. 237 00:09:06,360 --> 00:09:10,000 WE WANTED TO WORK WITH THE RDCC 238 00:09:10,000 --> 00:09:13,320 TO DO THIS AND FORWARD AS A 239 00:09:13,320 --> 00:09:13,920 COLLABORATION. 240 00:09:13,920 --> 00:09:15,560 WE WANT TO SEE WHAT NATURE OF 241 00:09:15,560 --> 00:09:17,120 THE ISSUE IS BEFORE WE GO OUT 242 00:09:17,120 --> 00:09:19,760 AND SAY THIS IS HARDLY MAY NOT 243 00:09:19,760 --> 00:09:20,440 BE THAT BAD. 244 00:09:20,440 --> 00:09:23,160 WHICH WOULD BE AWESOME OR IT 245 00:09:23,160 --> 00:09:23,800 THAT BAD. 246 00:09:23,800 --> 00:09:25,960 >> IF IT IS NOT YOU FIND ANOTHER 247 00:09:25,960 --> 00:09:27,520 AREA TO FOCUS ON THAT IS AND 248 00:09:27,520 --> 00:09:32,040 MOVE FORWARD. 249 00:09:32,040 --> 00:09:35,560 >> CAN EVERYONE HER HEAR US? 250 00:09:35,560 --> 00:09:35,960 >> YES. 251 00:09:35,960 --> 00:09:37,160 >> I HAVE BEEN WORKING WITH HIM 252 00:09:37,160 --> 00:09:40,720 FOR THREE AND A HALF YEARS. 253 00:09:40,720 --> 00:09:40,960 PRE-COVID. 254 00:09:40,960 --> 00:09:42,040 ON THAT. 255 00:09:42,040 --> 00:09:44,360 IT'S BEEN MORE INTERESTING 256 00:09:44,360 --> 00:09:45,640 PROJECTS I HAVE BEEN INVOLVED 257 00:09:45,640 --> 00:09:47,360 WITH IN A WHILE. 258 00:09:47,360 --> 00:09:48,560 TIINA URV IS ALSO INVOLVED AS 259 00:09:48,560 --> 00:09:51,360 WELCH 260 00:09:51,360 --> 00:09:52,680 >> GREAT, THANK YOU MARSHALL. 261 00:09:52,680 --> 00:09:57,480 GOOD TO KNOW. 262 00:09:57,480 --> 00:10:01,120 >> SO MIKE IS ACTIVELY RUNNING 263 00:10:01,120 --> 00:10:02,400 UPSTAIRS TO GIVE CONTROL OF THE 264 00:10:02,400 --> 00:10:04,320 SCREEN UNLESS SOMEBODY CAN DO IT 265 00:10:04,320 --> 00:10:06,040 FROM THERE THEN SHOULD BE GOOD 266 00:10:06,040 --> 00:10:11,280 TO GO. 267 00:10:11,280 --> 00:10:31,680 >> YES. 268 00:10:31,680 --> 00:10:40,000 >> PJ YOU NOW HAVE CONTROL. 269 00:10:40,000 --> 00:10:45,440 >> PERHAPS NOT. 270 00:10:45,440 --> 00:10:50,560 >> CAN THEY JUST DO IT FROM 271 00:10:50,560 --> 00:10:52,920 THEIR ROOM? 272 00:10:52,920 --> 00:10:54,360 MIC MAYBE OTHER SLIDES FROM HIS 273 00:10:54,360 --> 00:10:57,760 END. 274 00:10:57,760 --> 00:11:00,240 WHY WE SET IT UP THAT WAY. 275 00:11:00,240 --> 00:11:05,120 >> LET'S DO IT TO MAKE SURE. 276 00:11:05,120 --> 00:11:12,080 >> I WILL SAY NEXT SLIDE THEN. 277 00:11:12,080 --> 00:11:14,160 >> WE CAN HEAR YOU. 278 00:11:14,160 --> 00:11:20,400 >> NEXT SLIDE. 279 00:11:20,400 --> 00:11:21,160 >> ALL RIGHT. 280 00:11:21,160 --> 00:11:23,280 SO THIS IS A SLIDE ABOUT OUR 281 00:11:23,280 --> 00:11:28,480 MISSION WHICH IS TO TRY IMPROVE 282 00:11:28,480 --> 00:11:29,240 RARE DISEASE ENVIRONMENT FOR 283 00:11:29,240 --> 00:11:32,160 EVERYONE. 284 00:11:32,160 --> 00:11:34,200 WITH ALL ADVANCES IN SCIENCE OF 285 00:11:34,200 --> 00:11:37,160 LATE, WE FEEL THERE ARE FEW 286 00:11:37,160 --> 00:11:39,800 LIMITATIONS ON WHAT WE HAVE 287 00:11:39,800 --> 00:11:41,040 POTENTIAL TO DO. 288 00:11:41,040 --> 00:11:42,600 NEXT PLEASE. 289 00:11:42,600 --> 00:11:45,560 SO WHAT I'M -- WHAT WE WILL BE 290 00:11:45,560 --> 00:11:47,960 COVERING OVER THE NEXT SEVERAL 291 00:11:47,960 --> 00:11:49,880 MINUTES THERE ARE FEW POINTS TO 292 00:11:49,880 --> 00:11:51,960 KEEP IN MIND AS WE GO THROUGH 293 00:11:51,960 --> 00:11:52,440 THIS. 294 00:11:52,440 --> 00:11:56,720 WE HAVE MANY DISEASES, VERY FEW 295 00:11:56,720 --> 00:11:58,480 APPROVED TREATMENTS BUT WE ARE 296 00:11:58,480 --> 00:12:00,680 ALSO AT TIME OF UNPRECEDENTED 297 00:12:00,680 --> 00:12:01,760 SCIENTIFIC OPPORTUNITY WHICH WE 298 00:12:01,760 --> 00:12:04,280 TRYING TO TAKE ADVANTAGE OF. 299 00:12:04,280 --> 00:12:05,600 A LOT OF PROGRAMS ARE NOW TRYING 300 00:12:05,600 --> 00:12:08,240 TO THINK DIFFERENTLY, ADAPT OR 301 00:12:08,240 --> 00:12:09,800 ADOPT NEW STRATEGIES INCLUDING 302 00:12:09,800 --> 00:12:11,920 MANY DISEASES AT A TIME MODEL 303 00:12:11,920 --> 00:12:14,240 HERE DISEASE, DEFINE DISEASE 304 00:12:14,240 --> 00:12:16,240 DIFFERENTLY, OR PILOT 305 00:12:16,240 --> 00:12:17,120 NON-TRADITIONAL APPROACHES AND 306 00:12:17,120 --> 00:12:20,280 THAT IS WHAT WE WILL BE TOUCHING 307 00:12:20,280 --> 00:12:23,360 ON. 308 00:12:23,360 --> 00:12:24,600 THIS IS WHO WE ARE. 309 00:12:24,600 --> 00:12:28,000 AS YOU HEARD FROM JONI YESTERDAY 310 00:12:28,000 --> 00:12:30,480 I WILL BE STEPPING DOWN FROM 311 00:12:30,480 --> 00:12:32,440 NCATS AND PJ WILL TAKE OVER AS 312 00:12:32,440 --> 00:12:33,480 ACTING DIRECTOR. 313 00:12:33,480 --> 00:12:36,200 THE REST OF THE TEAM HOPEFULLY 314 00:12:36,200 --> 00:12:37,920 THEY LOOK FAMILIAR TO YOU BUT 315 00:12:37,920 --> 00:12:39,880 THIS IS A FANTASTIC GROUP OF 316 00:12:39,880 --> 00:12:41,560 PEOPLE WHO HAVE BEEN DOING GREAT 317 00:12:41,560 --> 00:12:41,960 WORK. 318 00:12:41,960 --> 00:12:45,600 NEXT PLEASE. 319 00:12:45,600 --> 00:12:47,680 SO WHAT ARE THE PROBLEM TRYING 320 00:12:47,680 --> 00:12:48,160 TO ADDRESS? 321 00:12:48,160 --> 00:12:51,160 RIGHT NOW THERE ARE ABOUT 10,000 322 00:12:51,160 --> 00:12:54,120 OR SO DIFFERENT RARE DISEASES 323 00:12:54,120 --> 00:12:55,360 THAT ARE KNOWN. 324 00:12:55,360 --> 00:12:57,760 THIS NUMBER INCREASES BY ABOUT 325 00:12:57,760 --> 00:12:59,160 200 TO 300 NEW DISEASES EACH 326 00:12:59,160 --> 00:13:03,000 YEAR. 327 00:13:03,000 --> 00:13:05,240 THE DISEASES ARE NOT NEW, THEY 328 00:13:05,240 --> 00:13:06,880 HAVE BEEN THERE ALL ALONG BUT 329 00:13:06,880 --> 00:13:08,440 OUR ABILITY TO RECOGNIZE HAS 330 00:13:08,440 --> 00:13:09,560 IMPROVED. 331 00:13:09,560 --> 00:13:12,720 90% OF 10,000 DISEASE FALL UNDER 332 00:13:12,720 --> 00:13:14,240 CATEGORY OF WHAT IS OFTEN CALLED 333 00:13:14,240 --> 00:13:16,960 ULTRA RARE SO THESE WOULD BE THE 334 00:13:16,960 --> 00:13:18,480 ONE IN A MILLION DISEASE OR 335 00:13:18,480 --> 00:13:19,960 LESS. 336 00:13:19,960 --> 00:13:21,480 SO THOSE WILL BE DISEASE THAT 337 00:13:21,480 --> 00:13:23,080 HAVE HUNDREDS TO A FEW THOUSANDS 338 00:13:23,080 --> 00:13:24,560 OF PATIENTS OR SOMETIMES EVEN 339 00:13:24,560 --> 00:13:26,920 FEWER THAN THAT. 340 00:13:26,920 --> 00:13:29,200 DIAGNOSIS REMAINS A REAL 341 00:13:29,200 --> 00:13:32,280 CHALLENGE, HARD TO DIAGNOSE 342 00:13:32,280 --> 00:13:34,800 PATIENTS, ESTIMATES ARE AROUND 5 343 00:13:34,800 --> 00:13:37,160 OR MORE YEARS TO OBTAIN ACCURATE 344 00:13:37,160 --> 00:13:39,720 DIAGNOSIS, SOMETIMES DECADES. 345 00:13:39,720 --> 00:13:41,160 WE KNOW RARE DISEASE PATIENTS 346 00:13:41,160 --> 00:13:41,960 ARE UNDERRECOGNIZED. 347 00:13:41,960 --> 00:13:45,960 WE KNOW THERE ARE PEOPLE WHO ARE 348 00:13:45,960 --> 00:13:46,760 UNDIAGNOSED. 349 00:13:46,760 --> 00:13:49,800 AND DON'T KNOW THEY HAVE A RARE 350 00:13:49,800 --> 00:13:50,320 DISEASE. 351 00:13:50,320 --> 00:13:52,600 THEY ARE THE MAJORITY OF 352 00:13:52,600 --> 00:13:54,160 DISEASES DON'T HAVE APPROVED 353 00:13:54,160 --> 00:13:55,720 THERAPY AND INCREASINGLY IT IS 354 00:13:55,720 --> 00:13:58,080 NOT NECESSARILY BECAUSE WE DON'T 355 00:13:58,080 --> 00:14:00,160 HAVE THE SCIENCE BEHIND IT BUT 356 00:14:00,160 --> 00:14:03,320 BECAUSE IT IS VERY HARD TO MAKE 357 00:14:03,320 --> 00:14:05,440 A BUSINESS MODEL ARGUMENT FOR A 358 00:14:05,440 --> 00:14:07,520 DISEASE THAT AFFECTS ONLY TEN 359 00:14:07,520 --> 00:14:07,880 PATIENTS. 360 00:14:07,880 --> 00:14:11,160 NEXT PLEASE. 361 00:14:11,160 --> 00:14:14,760 SO THIS IS INFORMED OUR THINKING 362 00:14:14,760 --> 00:14:17,040 AT ORDR, PROGRAMS AND 363 00:14:17,040 --> 00:14:18,720 INITIATIVES AND WHERE WE DECIDE 364 00:14:18,720 --> 00:14:19,800 WHERE WE WANT TO LOOK AND WHERE 365 00:14:19,800 --> 00:14:23,440 WE WANT TO TRY TO ADVANCE 366 00:14:23,440 --> 00:14:24,280 SCIENCE. 367 00:14:24,280 --> 00:14:27,280 THIS DROPS INTO THREE MAIN 368 00:14:27,280 --> 00:14:27,840 CATEGORIES. 369 00:14:27,840 --> 00:14:31,680 DATA AND INFORMATICS TO THE 370 00:14:31,680 --> 00:14:33,360 LEFT, RESEARCH PROJECTS IN THE 371 00:14:33,360 --> 00:14:35,160 MIDDLE AND WHAT WE TERM 372 00:14:35,160 --> 00:14:35,720 COLLABORATION. 373 00:14:35,720 --> 00:14:37,040 TO THE RIGHT. 374 00:14:37,040 --> 00:14:40,240 CAN YOU HIT NEXT? 375 00:14:40,240 --> 00:14:42,360 I'M GOING TO START DATA AND 376 00:14:42,360 --> 00:14:42,920 INFORMATICS. 377 00:14:42,920 --> 00:14:46,360 NEXT PLEASE. 378 00:14:46,360 --> 00:14:50,520 SO ONE OF OUR MOST IMPORTANT 379 00:14:50,520 --> 00:14:51,840 LONGEST STANDING PROGRAMS IS 380 00:14:51,840 --> 00:14:53,360 GENETICS AND RARE DISEASES 381 00:14:53,360 --> 00:14:55,920 INFORMATION CENTER OR GARD. 382 00:14:55,920 --> 00:14:59,000 THIS IS A CONGRESSIONALLY 383 00:14:59,000 --> 00:15:00,520 MANDATED PROGRAM WHERE IN THE 384 00:15:00,520 --> 00:15:03,400 RARE DISEASE ACT OF 2002 NIH WAS 385 00:15:03,400 --> 00:15:05,600 DIRECTED TO SET UP A QUOTE 386 00:15:05,600 --> 00:15:07,360 CLEARINGHOUSE ON RARE GENETIC 387 00:15:07,360 --> 00:15:10,200 DISEASE AND KEEPING IN MIND BACK 388 00:15:10,200 --> 00:15:12,480 IN 2002 WE KNEW A LOT LESS ABOUT 389 00:15:12,480 --> 00:15:14,000 GENETICS AND RARE DISEASE THAN 390 00:15:14,000 --> 00:15:15,920 WE DO NOW. 391 00:15:15,920 --> 00:15:20,640 SO THIS WAS FIRST STOOD UP AS 392 00:15:20,640 --> 00:15:21,320 INDIVIDUAL INCREASE SERVICE 393 00:15:21,320 --> 00:15:23,960 WHERE PEOPLE COULD CALL OR WRITE 394 00:15:23,960 --> 00:15:26,400 TO ASK QUESTIONS ABOUT RARE 395 00:15:26,400 --> 00:15:27,840 DISEASE, MOST COMMON PERSON 396 00:15:27,840 --> 00:15:29,080 CONTACTING US WOULD BE FOR 397 00:15:29,080 --> 00:15:30,760 EXAMPLE A PARENT OR PATIENT, 398 00:15:30,760 --> 00:15:32,280 JUST BEEN NEWLY DIAGNOSED AND 399 00:15:32,280 --> 00:15:34,080 DOESN'T KNOW WHAT TO DO. 400 00:15:34,080 --> 00:15:37,720 THEN THE WEBSITE WAS LAUNCHED IN 401 00:15:37,720 --> 00:15:37,920 2008. 402 00:15:37,920 --> 00:15:40,720 THE WEBSITE HAS FREELY 403 00:15:40,720 --> 00:15:42,560 ACCESSIBLE PATIENT FRIENDLY WEB 404 00:15:42,560 --> 00:15:44,960 PAGES ON 6500 RARE DISEASE AND 405 00:15:44,960 --> 00:15:48,560 WE STILL MAINTAIN THE INDIVIDUAL 406 00:15:48,560 --> 00:15:50,360 INQUIRY SERVICE SO MORE THAN 20 407 00:15:50,360 --> 00:15:52,160 MILLION USERS A YEAR, MORE THAN 408 00:15:52,160 --> 00:15:53,720 TEN THOUSAND INDIVIDUAL 409 00:15:53,720 --> 00:15:55,200 INQUIRIES EACH YEAR. 410 00:15:55,200 --> 00:15:57,000 THIS ACTUALLY ACCOUNTS FOR VERY 411 00:15:57,000 --> 00:15:59,840 HIGH PROPORTION OF NCATS WEB 412 00:15:59,840 --> 00:16:01,760 TRAFFIC AND HIGH PROPORTION OF 413 00:16:01,760 --> 00:16:03,480 NIH WEB TRAFFIC SO IF YOU CAME 414 00:16:03,480 --> 00:16:05,400 TO THE LANDING PAGE THIS IS WHAT 415 00:16:05,400 --> 00:16:07,040 IT WOULD LOOK LIKE AND PUT THE 416 00:16:07,040 --> 00:16:09,800 LINK THERE. 417 00:16:09,800 --> 00:16:12,280 SO WHAT IS NEW WITH GARD? 418 00:16:12,280 --> 00:16:12,840 A LOT. 419 00:16:12,840 --> 00:16:14,960 IT IS UNDERGOING A MAJOR 420 00:16:14,960 --> 00:16:17,360 REVISION TO GARD 2.0, WE HAVE 421 00:16:17,360 --> 00:16:18,360 BEEN WORKING ON THIS SEVERAL 422 00:16:18,360 --> 00:16:19,320 YEARS NOW. 423 00:16:19,320 --> 00:16:20,560 IT HAS BEEN A CHALLENGE TRYING 424 00:16:20,560 --> 00:16:23,000 TO KEEP UP WITH 6500 OR MORE 425 00:16:23,000 --> 00:16:25,360 RARE DISEASES AND TO TRY TO KEEP 426 00:16:25,360 --> 00:16:26,560 THAT INFORMATION UP TO DATE. 427 00:16:26,560 --> 00:16:28,360 IT IS REALLY ALMOST GONE BEYOND 428 00:16:28,360 --> 00:16:31,360 THE ABILITY FOR HUMANS TO DO 429 00:16:31,360 --> 00:16:32,960 THIS ALONE SO WE HAVE BEEN 430 00:16:32,960 --> 00:16:34,880 WORKING ON A MACHINE LEARNING 431 00:16:34,880 --> 00:16:36,560 MACHINE CAPTURE APPROACH AND 432 00:16:36,560 --> 00:16:38,600 ALSO TRY TO MAKE THE MOST OF THE 433 00:16:38,600 --> 00:16:40,400 INFORMATION THAT IS THERE. 434 00:16:40,400 --> 00:16:42,360 COLLECTED ONCE REUSE IT MANY 435 00:16:42,360 --> 00:16:44,040 TIMES, WHICH IS NOT SOMETHING 436 00:16:44,040 --> 00:16:45,760 THAT HAD BEEN IN THE OLDER 437 00:16:45,760 --> 00:16:47,040 MODEL. 438 00:16:47,040 --> 00:16:48,880 ANOTHER GOAL IS SINCE PATIENTS 439 00:16:48,880 --> 00:16:50,520 OFTEN HAVE TO ADVOCATE FOR THEIR 440 00:16:50,520 --> 00:16:52,720 RARE DISEASES IS TO TRY TO 441 00:16:52,720 --> 00:16:54,600 IMPROVE HEALTH LITERACY AROUND 442 00:16:54,600 --> 00:16:56,200 RARE GENETIC DISORDERS. 443 00:16:56,200 --> 00:16:58,960 SO WE REALLY HAVE EXCITING NEWS, 444 00:16:58,960 --> 00:17:03,240 THE GARD 2.0 SITE HAS DONE A 445 00:17:03,240 --> 00:17:05,040 TEST SITE LAUNCH. 446 00:17:05,040 --> 00:17:07,160 TO SHOW THAT TO YOU NOW. 447 00:17:07,160 --> 00:17:08,320 NEXT PLEASE. 448 00:17:08,320 --> 00:17:11,520 ON THE LEFT-HAND SIDE IS GARD 449 00:17:11,520 --> 00:17:13,600 1.0, THE OLD SITE, THAT PURPLE 450 00:17:13,600 --> 00:17:15,600 BOX THAT HAS CIRCLE AROUND IT, 451 00:17:15,600 --> 00:17:18,760 YOU CAN PREVIEW THE NEW SITE 452 00:17:18,760 --> 00:17:21,960 THAT WILL TAKE YOU TO GARD 2.0 453 00:17:21,960 --> 00:17:23,000 WHICH IS RIGHT. 454 00:17:23,000 --> 00:17:24,480 YOU CAN APPRECIATE THE DIFFERENT 455 00:17:24,480 --> 00:17:25,640 LOOK AND FEEL TO IT. 456 00:17:25,640 --> 00:17:27,800 THIS IS ON THE BACK END, A MUCH 457 00:17:27,800 --> 00:17:30,000 MORE MODULAR CONSTRUCTION WHERE 458 00:17:30,000 --> 00:17:33,200 THESE ARE DISCRETE LEARNING 459 00:17:33,200 --> 00:17:33,720 ELEMENTS. 460 00:17:33,720 --> 00:17:35,160 THE ADVANTAGE OF THIS IS WHEN 461 00:17:35,160 --> 00:17:37,800 YOU STORE IT THIS WAY YOU CAN 462 00:17:37,800 --> 00:17:39,760 REUSE IN NUMBER OF DIRECTIONS AS 463 00:17:39,760 --> 00:17:43,080 WELL AS TRYING TO MAKE THIS MORE 464 00:17:43,080 --> 00:17:44,840 USER FRIENDLY FOR THE PATIENTS. 465 00:17:44,840 --> 00:17:51,440 THIS IS AN EXAMPLE HERE CYSTIC 466 00:17:51,440 --> 00:17:53,640 FIBROSIS BOTH SIDES, LESS 467 00:17:53,640 --> 00:17:55,360 HORIZONTAL SCROLLING AND LENS 468 00:17:55,360 --> 00:17:58,720 DENSE TEXT AROUND MOVING TWO 469 00:17:58,720 --> 00:18:00,440 BOARDS LEARNING ELEMENTS TYPE 470 00:18:00,440 --> 00:18:03,960 MODULE THAT WOULD HOPEFULLY BE 471 00:18:03,960 --> 00:18:05,160 MORE MEANINGFUL AND ACTIONABLE 472 00:18:05,160 --> 00:18:06,000 TO PATIENTS. 473 00:18:06,000 --> 00:18:08,360 SO JUST ASKING IF YOU CAN ALL 474 00:18:08,360 --> 00:18:09,760 COME TAKE A LOOK AT THE SITE, 475 00:18:09,760 --> 00:18:12,160 LOOK AT THESE DISEASES AND WOULD 476 00:18:12,160 --> 00:18:17,560 REALLY WELCOME YOUR FEEDBACK. 477 00:18:17,560 --> 00:18:19,680 SOMETHING ELSE NEW, SINCE THE 478 00:18:19,680 --> 00:18:23,080 LAST TIME WE WERE HERE WE HAVE 479 00:18:23,080 --> 00:18:23,960 BEEN WORKING ON THE IDEA 480 00:18:23,960 --> 00:18:24,360 INITIATIVE. 481 00:18:24,360 --> 00:18:25,720 IT STANDS FOR IMPACT OF RARE 482 00:18:25,720 --> 00:18:27,080 DISEASE ON PATIENTS AND 483 00:18:27,080 --> 00:18:28,360 HEALTHCARE SYSTEMS A LITTLE BIT 484 00:18:28,360 --> 00:18:30,840 OF A TORTURED ACRONYM BUT WHAT 485 00:18:30,840 --> 00:18:33,040 WE ARE TRYING TO DO HERE IS TO 486 00:18:33,040 --> 00:18:35,880 QUANTIFY THE BURDEN OF RARE 487 00:18:35,880 --> 00:18:40,840 DISEASES TO TRY TO PUT SOME 488 00:18:40,840 --> 00:18:42,040 OBJECTIVE DATA AROUND THIS. 489 00:18:42,040 --> 00:18:44,560 SO TO DO THIS WE PERFORMED A 490 00:18:44,560 --> 00:18:45,200 PILOT STUDY. 491 00:18:45,200 --> 00:18:49,840 THIS WAS A COLLABORATION BETWEEN 492 00:18:49,840 --> 00:18:51,840 SEVERAL OF US HERE AT NCATS AS 493 00:18:51,840 --> 00:18:54,040 WELL AS UNIVERSITY OF COLORADO 494 00:18:54,040 --> 00:18:57,000 STANFORD HEALTH AND A DATA 495 00:18:57,000 --> 00:18:57,720 ANALYTICS COMPANY. 496 00:18:57,720 --> 00:19:01,320 NEXT PLEASE. 497 00:19:01,320 --> 00:19:03,560 TO THE LEFT OF THIS IS 498 00:19:03,560 --> 00:19:04,480 BEPUBLISHED A PAPER IN OCTOBER 499 00:19:04,480 --> 00:19:07,240 WITH THE RESULTS OF OUR STUDY 500 00:19:07,240 --> 00:19:10,680 AND WHAT THE STUDY DOES, IT WAS 501 00:19:10,680 --> 00:19:12,880 A 14 DISEASE PILOT, SELECTED 14 502 00:19:12,880 --> 00:19:15,520 REPRESENTATIVE DISEASES AND 503 00:19:15,520 --> 00:19:16,480 DELIBERATELY WORK THROUGH THIS 504 00:19:16,480 --> 00:19:18,520 TO QUANTIFY A FEW THINGS I WILL 505 00:19:18,520 --> 00:19:19,920 SHOW YOU IN A MINUTE. 506 00:19:19,920 --> 00:19:22,720 BOTTOM LINE ON THIS THREE WORDS 507 00:19:22,720 --> 00:19:24,480 TO THE RIGHT. 508 00:19:24,480 --> 00:19:26,600 RARE DISEASES ARE ACTUALLY 509 00:19:26,600 --> 00:19:30,160 COMMON, THEY ARE CERTAINLY VERY 510 00:19:30,160 --> 00:19:32,480 COSTLY AND THEY ARE POTENTIALLY 511 00:19:32,480 --> 00:19:34,160 ACTIONABLE. 512 00:19:34,160 --> 00:19:36,920 HERE ARE THE KEY FINDINGS FROM 513 00:19:36,920 --> 00:19:39,280 THE STUDY, SO WHAT WE DID IS 514 00:19:39,280 --> 00:19:41,560 TRIED TO FIND DISEASE WITHIN 515 00:19:41,560 --> 00:19:43,400 THESE VARIOUS EMR HEALTHCARE 516 00:19:43,400 --> 00:19:46,600 SYSTEM DATABASES. 517 00:19:46,600 --> 00:19:49,680 THE FIRST PASS WAS TO TRY TO 518 00:19:49,680 --> 00:19:50,480 IDENTIFY THE ICD CODE. 519 00:19:50,480 --> 00:19:51,920 THIS WAS CHALLENGING BECAUSE A 520 00:19:51,920 --> 00:19:53,960 LOT OF RARE DISEASES DON'T HAVE 521 00:19:53,960 --> 00:19:56,080 THEIR OWN ICD CODES OR THEY 522 00:19:56,080 --> 00:19:58,960 AREN'T CODED PROPERLY. 523 00:19:58,960 --> 00:20:01,520 SO WHAT WE SAW THERE THAT THIS 524 00:20:01,520 --> 00:20:03,960 WAS VERY HARD TO FIND AN 525 00:20:03,960 --> 00:20:04,880 ACCURATELY ESTIMATE NUMBER OF 526 00:20:04,880 --> 00:20:06,760 RARE DISEASE PATIENTS USING 527 00:20:06,760 --> 00:20:08,960 THESE READILY AVAILABLE DATA 528 00:20:08,960 --> 00:20:10,600 SOURCES TOOLS WE HAD SO WE HAD 529 00:20:10,600 --> 00:20:12,560 TO USE OTHER STAT STRATEGIES TO 530 00:20:12,560 --> 00:20:14,360 TRY TO FIND THE PATIENTS AND 531 00:20:14,360 --> 00:20:15,960 THEN QUANTIFY THINGS. 532 00:20:15,960 --> 00:20:17,560 THEN WHAT WE DID IS TRIED TO 533 00:20:17,560 --> 00:20:19,160 QUAWNTD FI THIS DIRECT MEDICAL 534 00:20:19,160 --> 00:20:21,720 COST OF RARE -- QUANTIFY THE 535 00:20:21,720 --> 00:20:23,120 DIRECT MEDICAL COST, THE COSTS 536 00:20:23,120 --> 00:20:24,360 ARE VERY HIGH. 537 00:20:24,360 --> 00:20:26,280 THIS IS A SURROGATE FOR ILLNESS 538 00:20:26,280 --> 00:20:28,160 RARE DISEASE TEND TO BE VERY 539 00:20:28,160 --> 00:20:29,640 SERIOUS AND RARE DISEASE 540 00:20:29,640 --> 00:20:32,800 PATIENTS ARE HIGH UTILIZERS OF 541 00:20:32,800 --> 00:20:33,640 MEDICAL HEALTHCARE SYSTEMS. 542 00:20:33,640 --> 00:20:35,520 WE WERE FINDING ABOUT THREE TO 543 00:20:35,520 --> 00:20:37,600 FIVE TIMES HIGHER COST FOR RARE 544 00:20:37,600 --> 00:20:40,400 DISEASE VERSUS NON-RARE 545 00:20:40,400 --> 00:20:42,760 CONTROLS, EXTRAPOLATING TO 25 TO 546 00:20:42,760 --> 00:20:44,200 30 RARE DISEASE PATIENTS THAT 547 00:20:44,200 --> 00:20:47,120 COMES TO SOMEWHERE AROUND $400 548 00:20:47,120 --> 00:20:47,760 BILLION A YEAR. 549 00:20:47,760 --> 00:20:50,560 WHAT WE ALSO DID IS WE TRIED TO 550 00:20:50,560 --> 00:20:51,800 QUANTIFY THAT DIAGNOSTIC 551 00:20:51,800 --> 00:20:54,080 ODYSSEY, THAT JOURNEY TO 552 00:20:54,080 --> 00:20:57,440 DIAGNOSIS, WE CAN SAY QUITE 553 00:20:57,440 --> 00:20:59,240 UNEQUIVOCALLY THAT DIAGNOSTIC 554 00:20:59,240 --> 00:21:01,200 JOURNEY IS REAL AND IT IS 555 00:21:01,200 --> 00:21:01,760 PROLONGED. 556 00:21:01,760 --> 00:21:04,240 AND IN SOME CASES RESULTS OF 557 00:21:04,240 --> 00:21:07,080 THIS DELAY RESULTS IN 558 00:21:07,080 --> 00:21:07,760 IRREVERSIBLE COMPLICATIONS THAT 559 00:21:07,760 --> 00:21:09,120 CAN CONTRIBUTE TO ONGOING HIGH 560 00:21:09,120 --> 00:21:11,200 COST OF CARE. 561 00:21:11,200 --> 00:21:12,880 WE WERE LOOKING FOR SIGNAL IN 562 00:21:12,880 --> 00:21:14,760 HERE WHERE MACHINE ASSISTED 563 00:21:14,760 --> 00:21:18,040 STRATEGIES MIGHT BE FEASIBLE. 564 00:21:18,040 --> 00:21:19,880 AND THIS IS A GOOD NEWS HERE. 565 00:21:19,880 --> 00:21:22,400 WE THINK THAT MAY BE POSSIBLE, 566 00:21:22,400 --> 00:21:24,920 THESE STRATEGIES NEED TO BE 567 00:21:24,920 --> 00:21:25,480 DEVELOPED. 568 00:21:25,480 --> 00:21:26,560 NEXT PLEASE. 569 00:21:26,560 --> 00:21:28,680 WE WEREN'T THE ONLY ONES THAT 570 00:21:28,680 --> 00:21:30,680 LOOKED AT THIS THE PAST YEAR, 571 00:21:30,680 --> 00:21:32,320 FOUR STUDIES PUBLISHED AROUND 572 00:21:32,320 --> 00:21:34,200 THE SAME TIME IN 2021. 573 00:21:34,200 --> 00:21:36,600 THEY WERE DONE VERY DIFFERENTLY 574 00:21:36,600 --> 00:21:37,760 BUT THEY CAME WITH THE SAME 575 00:21:37,760 --> 00:21:39,760 OVERALL MESSAGE, RARE DISEASES 576 00:21:39,760 --> 00:21:42,400 ARE COSTLY IN ECONOMIC AND HUMAN 577 00:21:42,400 --> 00:21:43,320 TERMS. 578 00:21:43,320 --> 00:21:45,280 HARD TO RECOGNIZE PATIENTS IN 579 00:21:45,280 --> 00:21:46,960 HEALTHCARE SYSTEM DATABASES BUT 580 00:21:46,960 --> 00:21:50,400 THE GOOD NEWS IS THEY ARE 581 00:21:50,400 --> 00:21:52,840 POTENTIALLY ACTIONABLE BUT 582 00:21:52,840 --> 00:21:53,440 UNDERPRIORITIZED GIVEN 583 00:21:53,440 --> 00:21:54,640 HEALTHCARE SYSTEM IMPACT. 584 00:21:54,640 --> 00:21:57,640 LISTED HERE THE OTHER STUDIES, 585 00:21:57,640 --> 00:21:59,640 THE LIFE FOUNDATION DID A BURDEN 586 00:21:59,640 --> 00:22:01,640 STUDY THEY LOOK AT DIRECT AND 587 00:22:01,640 --> 00:22:03,320 INDIRECT COST ABOUT $1 TRILLION 588 00:22:03,320 --> 00:22:06,800 A YEAR. 589 00:22:06,800 --> 00:22:09,480 OUR STUDIES CAME TO ABOUT 400 590 00:22:09,480 --> 00:22:10,680 BILLION WITH DIRECT COST REALLY 591 00:22:10,680 --> 00:22:12,920 NOT THAT DIFFERENT EVEN THOUGH 592 00:22:12,920 --> 00:22:15,960 IT USE DIFFERENT METHODOLOGIES 593 00:22:15,960 --> 00:22:18,040 AND AURORA HEALTH DID SOMETHING 594 00:22:18,040 --> 00:22:19,160 SIMILAR, CAME UP WITH SIMILAR 595 00:22:19,160 --> 00:22:19,400 NUMBERS. 596 00:22:19,400 --> 00:22:21,040 THERE WAS A GAO REPORT. 597 00:22:21,040 --> 00:22:24,200 THEY DID NOT QUANTIFY RARE 598 00:22:24,200 --> 00:22:25,760 DISEASE BUT DID COME TO THE 599 00:22:25,760 --> 00:22:27,560 CONCLUSION THAT AVAILABLE 600 00:22:27,560 --> 00:22:29,280 EVIDENCE SHOWS RARE DISEASES ARE 601 00:22:29,280 --> 00:22:31,280 VERY COSTLY. 602 00:22:31,280 --> 00:22:33,200 SO NO REAL SURPRISES THERE BUT I 603 00:22:33,200 --> 00:22:34,560 THINK IT WAS VERY IMPORTANT TO 604 00:22:34,560 --> 00:22:37,760 GET THIS OBJECTIVE INFORMATION. 605 00:22:37,760 --> 00:22:42,480 NEXT PLEASE. 606 00:22:42,480 --> 00:22:44,760 SO NOW JUST TOUCH ON SOME OF OUR 607 00:22:44,760 --> 00:22:50,360 RESEARCH PROGRAMS, NEXT PLEASE. 608 00:22:50,360 --> 00:22:53,240 THROUGH NEW THIS YEAR IS THE 609 00:22:53,240 --> 00:22:55,480 PUBLISHED FUNDING ANNOUNCEMENT 610 00:22:55,480 --> 00:22:57,760 ON TRYING TO DEVELOP STRATEGIES 611 00:22:57,760 --> 00:22:59,520 TO SHORTEN DIAGNOSTIC ODYSSEY. 612 00:22:59,520 --> 00:23:01,720 SO WE PUBLISHED A ONE TIME 613 00:23:01,720 --> 00:23:04,240 FUNDING ANNOUNCEMENT USING THE 614 00:23:04,240 --> 00:23:07,480 BIPHASIC MECHANISM. 615 00:23:07,480 --> 00:23:10,680 TO ASK APPLICANTS TO DEVELOP 616 00:23:10,680 --> 00:23:11,960 MULTI-DISCIPLINARY COMBINATION 617 00:23:11,960 --> 00:23:13,480 MACHINE ASSISTED GENOMIC 618 00:23:13,480 --> 00:23:14,720 ANALYSIS AND CLINICAL APPROACH 619 00:23:14,720 --> 00:23:19,360 TO TRY TO SHORTEN THAT ODYSSEY. 620 00:23:19,360 --> 00:23:21,840 SO WHAT WE ALSO ASKED THESE NOT 621 00:23:21,840 --> 00:23:23,960 BE APPLICABLE TO ONE OR TWO OR 622 00:23:23,960 --> 00:23:26,960 SMALL NUMBER OF RARE DISEASE BUT 623 00:23:26,960 --> 00:23:28,960 THESE STRATEGIES BE USED ACROSS 624 00:23:28,960 --> 00:23:31,040 A BROADER ARRAY OF RARE DISEASE 625 00:23:31,040 --> 00:23:32,800 TO TRY TO IDENTIFY PATIENTS 626 00:23:32,800 --> 00:23:33,800 FASTER AND VERY IMPORTANTLY WE 627 00:23:33,800 --> 00:23:35,800 THINK IS THIS A METHODOLOGY THAT 628 00:23:35,800 --> 00:23:38,760 COULD BE USED BY FRONT LINE 629 00:23:38,760 --> 00:23:40,560 PROVIDERS BECAUSE THAT IS OFTEN 630 00:23:40,560 --> 00:23:43,360 WHERE A LOT OF DELAY OCCURS. 631 00:23:43,360 --> 00:23:45,520 JUST ALSO LIKE TO MENTION THAT 632 00:23:45,520 --> 00:23:47,840 IN SEPTEMBER COUNCIL ALSO 633 00:23:47,840 --> 00:23:50,520 APPROVED SBIR MECHANISM TO TRY 634 00:23:50,520 --> 00:23:52,000 TO WORK ON THIS AS WELL. 635 00:23:52,000 --> 00:23:56,080 NEXT PLEASE. 636 00:23:56,080 --> 00:23:57,840 SO THIS WAS FEEDING INTO THIS 637 00:23:57,840 --> 00:24:00,160 LARGER QUESTION THAT WE 638 00:24:00,160 --> 00:24:01,760 IDENTIFIED IN IDEAS AS WELL AS 639 00:24:01,760 --> 00:24:04,840 TRYING TO WORK ON DIAGNOSTIC 640 00:24:04,840 --> 00:24:07,160 ODYSSEY, CAN WE DEVELOP WHAT WE 641 00:24:07,160 --> 00:24:08,880 ARE CALLING ZEBRA TRIGGERS. 642 00:24:08,880 --> 00:24:10,760 ARE THERE THINGS IN THE MEDICAL 643 00:24:10,760 --> 00:24:12,760 RECORD WE CAN USE RARE DISEASE 644 00:24:12,760 --> 00:24:14,560 INFORMATICS OR MACHINE LEARNING, 645 00:24:14,560 --> 00:24:16,400 TO TRY TO SHORTEN THIS APPROACH 646 00:24:16,400 --> 00:24:18,480 RATHER THAN JUST WHAT WE DO NOW 647 00:24:18,480 --> 00:24:20,640 IS BASICALLY GO PATIENT BY 648 00:24:20,640 --> 00:24:22,560 PATIENT TO TRY TO IDENTIFY 649 00:24:22,560 --> 00:24:22,960 PEOPLE. 650 00:24:22,960 --> 00:24:25,200 I'M SORRY, CLICK BACK AGAIN. 651 00:24:25,200 --> 00:24:28,160 SO WE ARE CALLING THESE ZEBRA 652 00:24:28,160 --> 00:24:30,360 TRIGGERS BECAUSE OF THE OLD 653 00:24:30,360 --> 00:24:32,240 MEDICAL SCHOOL ADAGE WHERE THEY 654 00:24:32,240 --> 00:24:36,320 TELL NEW MEDICAL STUDENTS THINGS 655 00:24:36,320 --> 00:24:38,200 COMMONLY FORCES NOT ZEBRAS. 656 00:24:38,200 --> 00:24:39,440 SO NEXT PLEASE. 657 00:24:39,440 --> 00:24:41,560 BUT WE ARE OF COURSE ZEBRAS SO 658 00:24:41,560 --> 00:24:43,640 WE WANT PEOPLE TO THINK ZEBRAS 659 00:24:43,640 --> 00:24:45,800 BUT WE WANT PEOPLE TO THINK 660 00:24:45,800 --> 00:24:46,960 ZEBRA SPECIFICALLY WHEN YOU 661 00:24:46,960 --> 00:24:48,680 START SEEING SOME OF WHAT WE 662 00:24:48,680 --> 00:24:51,160 THINK ARE SENTINEL 663 00:24:51,160 --> 00:24:52,760 CHARACTERISTICS SO YOUNGER AGES 664 00:24:52,760 --> 00:24:54,360 HIGHER UTILIZATION, MUST WANT PL 665 00:24:54,360 --> 00:24:55,520 CONCEPTS, TRAVELING ALL OVER 666 00:24:55,520 --> 00:25:01,640 TRYING TO GET DIAGNOSIS BUT 667 00:25:01,640 --> 00:25:03,400 SOMEBODY CALL IMPRECISE 668 00:25:03,400 --> 00:25:04,960 DIAGNOSTIC CODES SO WE MOSTLY 669 00:25:04,960 --> 00:25:07,560 DON'T HAVE ICD CODES, OUR IDEA 670 00:25:07,560 --> 00:25:09,160 STUDY IN THIS PRELIMINARY LOOK 671 00:25:09,160 --> 00:25:12,480 WE THINK THIS IS QUITE FEASIBLE. 672 00:25:12,480 --> 00:25:16,800 MENTION PLEASE. 673 00:25:16,800 --> 00:25:20,280 AND ANOTHER VERY LONG SANDING 674 00:25:20,280 --> 00:25:24,280 LARGEST PROGRAM RARE DISEASE 675 00:25:24,280 --> 00:25:26,040 RESEARCH NETWORK, THIS IS A 676 00:25:26,040 --> 00:25:27,480 NETWORK OF CENTERS OF EXCELLENCE 677 00:25:27,480 --> 00:25:29,320 THAT ARE GROUPED AROUND RARE 678 00:25:29,320 --> 00:25:31,000 DISEASE THERAPEUTIC AREAS AND 679 00:25:31,000 --> 00:25:35,160 THE PURPOSE OF THE RDCRN IS 680 00:25:35,160 --> 00:25:38,160 FACILITATE COLLABORATIVE 681 00:25:38,160 --> 00:25:38,880 RESEARCH. 682 00:25:38,880 --> 00:25:41,440 THE NETWORK IS GROUPED TO 683 00:25:41,440 --> 00:25:43,320 CONSORTIA AND THEY HAVE TO STUDY 684 00:25:43,320 --> 00:25:45,160 THREE OR MORE DISEASES RELATED 685 00:25:45,160 --> 00:25:47,720 IN SOME WAY SO FOR EXAMPLE, 686 00:25:47,720 --> 00:25:51,160 COULD BE A RARE LUNG CONSORTIA 687 00:25:51,160 --> 00:25:55,000 OR RARE MITOCHONDRIAL CONSORTIA 688 00:25:55,000 --> 00:25:58,880 OR MUTATION OR SOME OTHER FEE 689 00:25:58,880 --> 00:26:01,600 AROUND WHICH THE CONSORTIA IS 690 00:26:01,600 --> 00:26:02,360 GROUPED. 691 00:26:02,360 --> 00:26:04,440 IN THIS PATIENTS RESEARCH AND 692 00:26:04,440 --> 00:26:06,720 CLINICIANS ALL WORK TOGETHER SO 693 00:26:06,720 --> 00:26:08,960 THIS IS FIRST ESTABLISHED IN 694 00:26:08,960 --> 00:26:11,440 2002, THESE ARE FIVE YEAR 695 00:26:11,440 --> 00:26:13,960 AWARDEE COHORT AND WE ARE RIGHT 696 00:26:13,960 --> 00:26:16,360 NOW GETTING TOWARDS THE END OF 697 00:26:16,360 --> 00:26:19,000 WHAT ARE THE FOURTH COHORT WHICH 698 00:26:19,000 --> 00:26:21,480 IS SCHEDULED TO END IN 2023. 699 00:26:21,480 --> 00:26:25,760 SO CURRENTLY THE RDCRN INCLUDES 700 00:26:25,760 --> 00:26:27,400 20 CONSORTIA, I WILL MENTION 701 00:26:27,400 --> 00:26:29,680 DATA COORDINATING CENTER AND IT 702 00:26:29,680 --> 00:26:30,840 IS TEN NIH INSTITUTES AND 703 00:26:30,840 --> 00:26:33,440 CENTERS THAT COLLABORATE IN THE 704 00:26:33,440 --> 00:26:35,960 RDCRN. 705 00:26:35,960 --> 00:26:37,920 THIS IS A BRIEF OVERVIEW OF THE 706 00:26:37,920 --> 00:26:40,040 CURRENT RDCRN AND THROUGH THIS 707 00:26:40,040 --> 00:26:42,960 WE ARE ABLE TO STUDY ALMOST 200 708 00:26:42,960 --> 00:26:44,760 DIFFERENT RARE DISEASES, MORE 709 00:26:44,760 --> 00:26:47,000 THAN 300 CLINICAL SITES, 160 710 00:26:47,000 --> 00:26:49,080 PATIENT GROUPS AND MANY PATIENTS 711 00:26:49,080 --> 00:26:50,520 STUDIES AND TRIALS. 712 00:26:50,520 --> 00:26:54,320 SO SEEING MORE ABOUT THIS AND 713 00:26:54,320 --> 00:26:55,400 CONCEPT PRESENTATION THAT WILL 714 00:26:55,400 --> 00:26:58,280 FOLLOW THIS, RETURN TO THIS IN A 715 00:26:58,280 --> 00:26:58,880 LITTLE WHILE. 716 00:26:58,880 --> 00:27:01,360 NEXT PLEASE. 717 00:27:01,360 --> 00:27:04,560 HERE IS A FEW CUMULATIVE STATS 718 00:27:04,560 --> 00:27:06,360 FROM THE RDCRN. 719 00:27:06,360 --> 00:27:08,440 THIS IS A SUCCESSFUL MODEL MANY 720 00:27:08,440 --> 00:27:09,680 DISEASES AT TIME OF APPROACH FOR 721 00:27:09,680 --> 00:27:11,720 RARE DISEASE WHERE WE ARE ABLE 722 00:27:11,720 --> 00:27:14,040 TO LEARN FROM ONE ANOTHER ACROSS 723 00:27:14,040 --> 00:27:15,520 DISEASE AND WITHIN DISEASE 724 00:27:15,520 --> 00:27:18,160 CLUSTERS. 725 00:27:18,160 --> 00:27:19,800 THE NETWORK SINCE INCEPTION HAS 726 00:27:19,800 --> 00:27:22,360 HAD MORE THAN 1500 PUBLICATIONS, 727 00:27:22,360 --> 00:27:24,160 THOUSANDS OF PRESENTATIONS AND 728 00:27:24,160 --> 00:27:26,560 SUPPORTED MORE THAN 300 EARLY 729 00:27:26,560 --> 00:27:27,920 STAGE INVESTIGATORS. 730 00:27:27,920 --> 00:27:31,880 WE DID THE STATS OF THE STUDIES, 731 00:27:31,880 --> 00:27:33,200 87 NATURAL HISTORY STUDIES WHICH 732 00:27:33,200 --> 00:27:34,440 IS A LOT. 733 00:27:34,440 --> 00:27:35,760 CERTAINLY FOR RARE DISEASES SOME 734 00:27:35,760 --> 00:27:39,080 OF THESE HAVE BEEN GOING ON FOR 735 00:27:39,080 --> 00:27:41,600 MORE THAN 15 YEARS, JUST A 736 00:27:41,600 --> 00:27:45,320 REALLY GOOD BODY OF INFORMATION. 737 00:27:45,320 --> 00:27:47,160 STUDIES THAT 90 STUDIES DEVELOP 738 00:27:47,160 --> 00:27:50,360 OUTCOME MEASURES THAT CURRENTLY 739 00:27:50,360 --> 00:27:55,200 HAVE 26 ONGOING, 28 -- 38 740 00:27:55,200 --> 00:27:56,600 COMPLETE CLINICAL TRIALS THINGS 741 00:27:56,600 --> 00:27:58,800 THAT -- FOUR LED TO DRUG 742 00:27:58,800 --> 00:28:01,120 APPROVALS AND ABOUT 76 HAVE LED 743 00:28:01,120 --> 00:28:02,320 TO SOME KIND OF CHANGE IN 744 00:28:02,320 --> 00:28:03,040 CLINICAL PRACTICE. 745 00:28:03,040 --> 00:28:05,360 SO THIS IS REMARKABLE GIVEN THE 746 00:28:05,360 --> 00:28:07,120 LANDSCAPE OF RARE DISEASES. 747 00:28:07,120 --> 00:28:09,200 I THINK IT IS DEFINITELY SHOWING 748 00:28:09,200 --> 00:28:09,720 VALUE. 749 00:28:09,720 --> 00:28:12,760 NEXT PLEASE. 750 00:28:12,760 --> 00:28:16,040 ANOTHER NEWER CATEGORY OF GRANTS 751 00:28:16,040 --> 00:28:18,560 IS CLINICAL TRIAL READINESS 752 00:28:18,560 --> 00:28:18,960 GRANTS. 753 00:28:18,960 --> 00:28:20,560 THIS HAS BEEN A THREE YEAR 754 00:28:20,560 --> 00:28:22,560 PROGRAMMATIC ANNOUNCEMENT 755 00:28:22,560 --> 00:28:25,400 BETWEEN 2018 AND 2021 FOR TWO 756 00:28:25,400 --> 00:28:28,720 TYPES OF GRANTS, THE R21 757 00:28:28,720 --> 00:28:30,800 INNOVATION GRANTS OR THE RO 3 758 00:28:30,800 --> 00:28:31,280 SMALL GRANTS. 759 00:28:31,280 --> 00:28:33,600 THE PURPOSE OF THIS IS TO TRY TO 760 00:28:33,600 --> 00:28:35,080 ENABLE EFFICIENT AND EFFECTIVE 761 00:28:35,080 --> 00:28:36,520 MOVEMENT OF CANDIDATE 762 00:28:36,520 --> 00:28:38,720 THERAPEUTIC OR DIAGNOSTICS 763 00:28:38,720 --> 00:28:40,080 TOWARDS CLINICAL TRIALS AND 764 00:28:40,080 --> 00:28:41,640 PARTICULARLY FOCUSING ON THINGS 765 00:28:41,640 --> 00:28:44,120 SUCH AS BIOMARKERS AND CLINICAL 766 00:28:44,120 --> 00:28:48,240 OUTCOME ASSESSMENTS TRYING TO 767 00:28:48,240 --> 00:28:48,960 VALIDATE THOSE. 768 00:28:48,960 --> 00:28:51,280 IT ALSO INCLUDE TRYING TO BETTER 769 00:28:51,280 --> 00:28:53,400 DEFINE A RARE DISEASE SUCH AS 770 00:28:53,400 --> 00:28:56,120 THROUGH DATA ANALYTICS OF THESE 771 00:28:56,120 --> 00:28:57,120 NATURAL HISTORY TRIALS OR 772 00:28:57,120 --> 00:28:58,360 DEVELOP ANIMAL MODELS. 773 00:28:58,360 --> 00:29:00,960 SO WE CAME OUT WITH THIS THREE 774 00:29:00,960 --> 00:29:02,080 YEARS AGO IT WAS THE SUGGESTION 775 00:29:02,080 --> 00:29:03,640 OF RARE DISEASE RESEARCH VAI 776 00:29:03,640 --> 00:29:04,840 TOFERS WHO NOTICE -- 777 00:29:04,840 --> 00:29:06,160 INVESTIGATOR WHOSE NOTICED THIS 778 00:29:06,160 --> 00:29:06,760 AS A GAP. 779 00:29:06,760 --> 00:29:11,400 SO WE MADE OVER 25 AWARDS, NICHD 780 00:29:11,400 --> 00:29:14,840 HAS SIGNED ON AND I THINK IT IS 781 00:29:14,840 --> 00:29:16,160 REALLY ADDRESS PROBLEM IN A 782 00:29:16,160 --> 00:29:18,800 BROADWAY, VERY DIVERSE BROAD 783 00:29:18,800 --> 00:29:20,760 ARRAY OF RARE DISEASE THAT HAVE 784 00:29:20,760 --> 00:29:22,600 BEEN INVOLVED IN THESE AWARDS AS 785 00:29:22,600 --> 00:29:24,880 WELL AS VERY DIFFERENT 786 00:29:24,880 --> 00:29:28,800 APPROACHES TO TRY TO INCLUDE -- 787 00:29:28,800 --> 00:29:31,560 IMPROVE THAT CLINICAL TRIAL 788 00:29:31,560 --> 00:29:34,360 READINESS. 789 00:29:34,360 --> 00:29:37,360 AT THIS POINT IN TIME I'D HAND 790 00:29:37,360 --> 00:29:39,960 IT OVER. 791 00:29:39,960 --> 00:29:41,040 >> THANKS, ANNE. 792 00:29:41,040 --> 00:29:44,960 I WILL TAKE IT UP HERE WITHIN 793 00:29:44,960 --> 00:29:45,960 RESEARCH PRESENTATION AND TALK 794 00:29:45,960 --> 00:29:48,160 ABOUT VARIETY OF OUR MANY 795 00:29:48,160 --> 00:29:50,280 DISEASE AT TIME APPROACHES AND 796 00:29:50,280 --> 00:29:50,840 PROJECTS. 797 00:29:50,840 --> 00:29:53,440 NEXT SLIDE. 798 00:29:53,440 --> 00:29:56,840 THE BASIS OF THIS IS FOR ONE OF 799 00:29:56,840 --> 00:30:00,120 THESE WE CALL FOCUSED ON SHARED 800 00:30:00,120 --> 00:30:02,000 MOLECULAR ETIOLOGIES OR THR 801 00:30:02,000 --> 00:30:02,560 PEUTICS. 802 00:30:02,560 --> 00:30:04,840 IT HAS TO DO WITH THE FACT THAT 803 00:30:04,840 --> 00:30:07,040 DISEASES INCLUDING RARE DISEASE 804 00:30:07,040 --> 00:30:09,440 IDENTIFY AND TREATED BASED ON 805 00:30:09,440 --> 00:30:10,560 CLINICAL MANIFESTATIONS 806 00:30:10,560 --> 00:30:11,440 PHENOTYPE SO WE HAVE THOUSANDS 807 00:30:11,440 --> 00:30:13,360 OF RARE GENETIC DISEASE BUT THE 808 00:30:13,360 --> 00:30:16,760 GOOD NEWS IS WE HAVE FAR FEWER 809 00:30:16,760 --> 00:30:18,760 UNDERLYING ETIOLOGIES, HERE 810 00:30:18,760 --> 00:30:19,800 TALKING GENETIC DISEASE AND 811 00:30:19,800 --> 00:30:21,520 SYSTEM OF THESE INCLUDE 812 00:30:21,520 --> 00:30:23,960 PREMATURE STOP CODONS AND MISS 813 00:30:23,960 --> 00:30:27,320 FOLDED PROTEINS, SIGNAL DEFECTS, 814 00:30:27,320 --> 00:30:29,320 ET CETERA. 815 00:30:29,320 --> 00:30:30,600 WE DEVELOP THERAPEUTICS TO 816 00:30:30,600 --> 00:30:32,360 TARGET THESE ETIOLOGIES. 817 00:30:32,360 --> 00:30:35,080 SO THE IDEA IS GROUP DISEASE IN 818 00:30:35,080 --> 00:30:36,800 NON-TRADITIONAL WAYS, TO PAY 819 00:30:36,800 --> 00:30:39,360 LARGER PATIENT POOLS AND GAIN 820 00:30:39,360 --> 00:30:42,000 EFFICIENCIES IN DEVELOPMENT AND 821 00:30:42,000 --> 00:30:42,600 PARTICULARLY CLINICAL TRIALS 822 00:30:42,600 --> 00:30:45,320 START UP. 823 00:30:45,320 --> 00:30:46,000 NEXT SLIDE. 824 00:30:46,000 --> 00:30:48,600 SO THIS SHOWS A CARTOON EXAMPLE 825 00:30:48,600 --> 00:30:50,200 OF THIS, WE HAVE FOUR DIFFERENT 826 00:30:50,200 --> 00:30:52,640 DISEASES HERE, A B C AND D. 827 00:30:52,640 --> 00:30:53,960 WITHIN EACH DISEASE THERE ARE 828 00:30:53,960 --> 00:30:55,160 PATIENTS WITH DIFFERENT 829 00:30:55,160 --> 00:30:57,200 UNDERLYING ETIOLOGIES AND SO WE 830 00:30:57,200 --> 00:30:58,560 CAN IMAGINE IF WE HAD A DRUG 831 00:30:58,560 --> 00:31:00,640 THAT TARGETS THE ETIOLOGY IN RED 832 00:31:00,640 --> 00:31:02,480 FOR EXAMPLE, WHAT IS THE BEST 833 00:31:02,480 --> 00:31:04,280 WAY TO VIEW A CLINICAL TRIAL? 834 00:31:04,280 --> 00:31:06,160 AND THE WAY TO DO IT WOULD BE 835 00:31:06,160 --> 00:31:07,800 PULL OUT THOSE PATIENTS, 836 00:31:07,800 --> 00:31:10,040 IDENTIFY THOSE PATIENTS WITH THE 837 00:31:10,040 --> 00:31:15,120 RED ETIOLOGY, PUT THEM ALL 838 00:31:15,120 --> 00:31:18,360 TOGETHER INTO A NEW GROUP. 839 00:31:18,360 --> 00:31:21,320 AND THEN DO THE CLINICAL TRIAL 840 00:31:21,320 --> 00:31:22,800 ON THOSE INDIVIDUALS USING THE 841 00:31:22,800 --> 00:31:24,280 SAME DRUG AND FIGURE OUT WHAT 842 00:31:24,280 --> 00:31:25,960 THE OUTCOME MEASURES ARE. 843 00:31:25,960 --> 00:31:27,440 ESSENTIALLY PUT THOSE PATIENTS 844 00:31:27,440 --> 00:31:30,480 INTO A BASKET AND HENCE THE TERM 845 00:31:30,480 --> 00:31:32,280 BASKET TRIALS WHERE YOU HAVE 846 00:31:32,280 --> 00:31:34,040 DIFFERENT DISEASES AND ONE 847 00:31:34,040 --> 00:31:34,600 THERAPEUTIC. 848 00:31:34,600 --> 00:31:35,120 NEXT SLIDE. 849 00:31:35,120 --> 00:31:37,640 AND THE GOOD NEWS HERE IS THAT 850 00:31:37,640 --> 00:31:39,320 THERE IS A PRECEDENT FOR THIS 851 00:31:39,320 --> 00:31:40,560 IMPORTANTLY A REGULATORY 852 00:31:40,560 --> 00:31:41,280 PRECEDENT. 853 00:31:41,280 --> 00:31:43,560 THESE ARE GENOMICALLY DRIVEN 854 00:31:43,560 --> 00:31:44,480 ONCOLOGY BASKET TRIALS AND HERE 855 00:31:44,480 --> 00:31:47,720 IS AN EXAMPLE OF A DRUG THAT 856 00:31:47,720 --> 00:31:49,760 TARGETS A SHARED ETIOLOGY, THE 857 00:31:49,760 --> 00:31:51,480 CLINICAL TRIAL THAT INVOLVED 858 00:31:51,480 --> 00:31:52,800 MANY DIFFERENT TYPES OF TUMORS. 859 00:31:52,800 --> 00:31:54,280 YOU CAN SEE THE NUMBERS HERE 860 00:31:54,280 --> 00:31:56,240 VERY SMALL NUMBERS NOBODY ON THE 861 00:31:56,240 --> 00:31:57,920 RIGHT MIND WOULD HAVE START AD 862 00:31:57,920 --> 00:32:01,560 WHOLE CLINICAL TRIAL FOR 863 00:32:01,560 --> 00:32:03,280 CARCINOMAS WITH THIS DRUG BUT 864 00:32:03,280 --> 00:32:04,760 PUTTING THEM TOGETHER IN A 865 00:32:04,760 --> 00:32:06,200 BASKET MAKES VERY EFFICIENT 866 00:32:06,200 --> 00:32:07,120 CLINICAL TRIAL AND VERY 867 00:32:07,120 --> 00:32:08,800 IMPORTANTLY LED TO FDA APPROVAL 868 00:32:08,800 --> 00:32:11,680 OF THIS DRUG. 869 00:32:11,680 --> 00:32:13,480 WHAT WE WANT TO DO IS TAKE THIS 870 00:32:13,480 --> 00:32:16,960 APPROACH AND PLY TO RARE DISEASE 871 00:32:16,960 --> 00:32:20,760 FOR DRUGS THAT SHARE M MOLECULAR 872 00:32:20,760 --> 00:32:22,720 ETIOLOGIES SO WE PUT OUT A 873 00:32:22,720 --> 00:32:23,720 FUNDING OPPORTUNITY FOR THIS, WE 874 00:32:23,720 --> 00:32:26,040 FUNDED TWO PROJECTS AS SHOWN 875 00:32:26,040 --> 00:32:26,520 THERE. 876 00:32:26,520 --> 00:32:28,800 ONE OF THOSE THE LATTER ONE WE 877 00:32:28,800 --> 00:32:30,360 TALK ABOUT JAK STAT GAIN OF 878 00:32:30,360 --> 00:32:31,920 FUNCTION DISORDERS IS A REALLY 879 00:32:31,920 --> 00:32:33,800 GREAT EXAMPLE OF DIFFERENT TYPE 880 00:32:33,800 --> 00:32:34,800 OF DISEASE THAT WE CAN THINK 881 00:32:34,800 --> 00:32:38,320 ABOUT THAT'S FOCUSED ON A SHARED 882 00:32:38,320 --> 00:32:38,960 ETIOLOGY. 883 00:32:38,960 --> 00:32:40,840 SO THESE ARE ONGOING AND AS I 884 00:32:40,840 --> 00:32:46,360 MENTION IN THE CHAT YESTERDAY 885 00:32:46,360 --> 00:32:48,680 THE BIG FOCUS ON THESE IS FOCUS 886 00:32:48,680 --> 00:32:50,240 ON REGULATORY PROCESS. 887 00:32:50,240 --> 00:32:52,560 THE MILESTONES, BIG MILESTONE IS 888 00:32:52,560 --> 00:32:54,320 GETTING AN IND FOR A CLINICAL 889 00:32:54,320 --> 00:32:57,720 TRIAL LIKE THIS, AND DISCUSSIONS 890 00:32:57,720 --> 00:32:59,960 AND INTERACTION WITH THE FDA 891 00:32:59,960 --> 00:33:01,880 REQUIRED AS PART OF THIS AWARD. 892 00:33:01,880 --> 00:33:03,760 A KEY THING HERE SOMETHING WE 893 00:33:03,760 --> 00:33:05,400 ALSO DO IN WRITING IS WE 894 00:33:05,400 --> 00:33:07,320 ENCOURAGE INVESTIGATORS TO 895 00:33:07,320 --> 00:33:10,160 CHOOSE DISEASE WHETHER MULTIPLE 896 00:33:10,160 --> 00:33:11,520 AND DIVERSE CLINICAL OUTCOMES 897 00:33:11,520 --> 00:33:13,800 BECAUSE THE ONE ADVANCE -- 898 00:33:13,800 --> 00:33:16,920 ADVANTAGE THAT CANCER HAS IS 899 00:33:16,920 --> 00:33:18,520 BASICALLY FOR ONCOLOGY STUDY IT 900 00:33:18,520 --> 00:33:20,360 IS OUTCOMES IS BASICALLY DOES 901 00:33:20,360 --> 00:33:22,560 THE TUMOR GET BIGGER SMALLER 902 00:33:22,560 --> 00:33:23,480 STAY THE SAME. 903 00:33:23,480 --> 00:33:25,760 RARE DISEASES THAT THE OUTCOMES 904 00:33:25,760 --> 00:33:28,080 ARE GOING TO BE DIVERSE, WE WANT 905 00:33:28,080 --> 00:33:30,280 TO INVESTIGATORS TO FIGURE OUT 906 00:33:30,280 --> 00:33:32,160 AND GRAPPLE WITH THAT CHALLENGE 907 00:33:32,160 --> 00:33:33,360 AND DESIGN CLINICAL TRIAL 908 00:33:33,360 --> 00:33:34,280 GETTING THE IND. 909 00:33:34,280 --> 00:33:36,960 SO THESE ARE JUST STARTING OUT 910 00:33:36,960 --> 00:33:39,040 NOW VERY INTERESTING TO SEE 911 00:33:39,040 --> 00:33:39,800 WHERE THEY GO. 912 00:33:39,800 --> 00:33:40,760 WE HAVE ANOTHER FUNDING 913 00:33:40,760 --> 00:33:42,000 OPPORTUNITY THAT SUPPORT IT IS 914 00:33:42,000 --> 00:33:45,160 SAME THING THAT IS FOR SMALL 915 00:33:45,160 --> 00:33:47,200 BUSINESSES TO THE SBIR 916 00:33:47,200 --> 00:33:48,360 MECHANISM. 917 00:33:48,360 --> 00:33:51,040 WE ALSO PARTICIPATE THIS WE 918 00:33:51,040 --> 00:33:52,480 CAN'T TALK A LOT ABOUT THIS 919 00:33:52,480 --> 00:33:53,760 BECAUSE OF TIME BUT WE 920 00:33:53,760 --> 00:33:55,800 PARTICIPATE IN THE INTERNATIONAL 921 00:33:55,800 --> 00:33:58,360 RARE DISEASE RESEARCH CONSORTIUM 922 00:33:58,360 --> 00:34:01,440 AND WE STARTED UP A WORKING 923 00:34:01,440 --> 00:34:03,320 GROUP TO FOCUS ON THE SAME ISSUE 924 00:34:03,320 --> 00:34:06,760 OF DRUGS AND CLINICAL TRIALS 925 00:34:06,760 --> 00:34:07,880 DRRGS TARGETING SHARED 926 00:34:07,880 --> 00:34:09,000 ETIOLOGIES AND MULTIPLE RARE 927 00:34:09,000 --> 00:34:12,760 DISEASES. 928 00:34:12,760 --> 00:34:14,200 NEXT SLIDE. 929 00:34:14,200 --> 00:34:15,760 MEETING TOWARDS GENETHER PE AND 930 00:34:15,760 --> 00:34:17,560 GENE EDITING WHICH ARE CLEARLY 931 00:34:17,560 --> 00:34:19,240 THERAPEUTIC PLATFORMS APPLICABLE 932 00:34:19,240 --> 00:34:22,560 TO MULTIPLE DISEASE, JONI TALKED 933 00:34:22,560 --> 00:34:24,080 LAST COUNCIL ABOUT PLATFORM 934 00:34:24,080 --> 00:34:28,080 VECTOR GENE THERAPIES OR PEGT 935 00:34:28,080 --> 00:34:29,880 PROGRAM, THAT IS A COLLABORATIVE 936 00:34:29,880 --> 00:34:33,640 EFFORT BETWEEN ORDR, NCATS 937 00:34:33,640 --> 00:34:37,720 THERAPEUTICS DEVELOPMENT BRANCH. 938 00:34:37,720 --> 00:34:40,240 NCATS OFFICE OF STRATEGIC 939 00:34:40,240 --> 00:34:42,480 INVESTIGATORS AND NHGRI AND 940 00:34:42,480 --> 00:34:42,960 NINDS. 941 00:34:42,960 --> 00:34:45,880 THIS IS A PUBLIC PLATFORM GENE 942 00:34:45,880 --> 00:34:48,280 THERAPY TRIAL, WE ARE TRYING TO 943 00:34:48,280 --> 00:34:50,160 STARTUP CLINICAL TRIALS FOR FOUR 944 00:34:50,160 --> 00:34:51,960 DISEASES, USING THE SAME 945 00:34:51,960 --> 00:34:54,480 THERAPEUTIC PLATFORM, SAME AAV 946 00:34:54,480 --> 00:34:56,440 VECTOR, SAME ROUTE OF 947 00:34:56,440 --> 00:34:58,000 ADMINISTRATION, SAME PRODUCTION 948 00:34:58,000 --> 00:34:59,760 FEURTYCATION, THE PROCESS AS A 949 00:34:59,760 --> 00:35:02,120 PRODUCT AS HAY SAY AD FDA. 950 00:35:02,120 --> 00:35:05,680 AND JUST CHANGING THERAPEUTIC 951 00:35:05,680 --> 00:35:07,200 PAYLOAD INTO THOSE GENE 952 00:35:07,200 --> 00:35:07,880 THERAPIES. 953 00:35:07,880 --> 00:35:10,560 I THINK WHAT MAKES THIS PROGRAM 954 00:35:10,560 --> 00:35:12,560 DIFFERENT AND UNIQUE IS WE PLAN 955 00:35:12,560 --> 00:35:14,200 TO MAKE ALL THE BROADLY RELEVANT 956 00:35:14,200 --> 00:35:15,440 DATA INCLUDING OUR 957 00:35:15,440 --> 00:35:17,200 COMMUNICATIONS WITH THE FDA 958 00:35:17,200 --> 00:35:18,760 PUBLICLY AVAILABLE SO OTHER 959 00:35:18,760 --> 00:35:20,320 PEOPLE CAN LEARN FROM IT AND 960 00:35:20,320 --> 00:35:22,760 REALLY THINKING HERE A LOT ABOUT 961 00:35:22,760 --> 00:35:25,440 THE FAMILIES AND OF PATIENTS 962 00:35:25,440 --> 00:35:27,720 WITH RARE DISEASES THAT ARE TOO 963 00:35:27,720 --> 00:35:29,520 RARE TO GAIN COMMERCIAL INTEREST 964 00:35:29,520 --> 00:35:31,080 AND SO ALL THESE FAMILIES ARE 965 00:35:31,080 --> 00:35:35,040 HAVING TO START UP THESE AAV 966 00:35:35,040 --> 00:35:36,000 THERAPY GENE CLINICAL TRIALS ON 967 00:35:36,000 --> 00:35:36,600 THEIR OWN. 968 00:35:36,600 --> 00:35:37,960 YOU WOULD LIKE TO MAKE THIS 969 00:35:37,960 --> 00:35:39,960 WHOLE PROCESS EASIER FOR THEM. 970 00:35:39,960 --> 00:35:42,400 NEXT SLIDE. 971 00:35:42,400 --> 00:35:46,560 IT IS BASED UPON THE FACT THAT 972 00:35:46,560 --> 00:35:49,880 ADENOASSOCIATED VIRUSSER AAV CAN 973 00:35:49,880 --> 00:35:52,080 BE USED IN FAIRLY STRAIGHT 974 00:35:52,080 --> 00:35:52,920 FORWARD WAY AT LEAST 975 00:35:52,920 --> 00:35:54,320 TECHNICALLY, THE WAY THAT WORKS 976 00:35:54,320 --> 00:35:57,600 IS WE TAKE THE AAV 977 00:35:57,600 --> 00:35:59,560 ADENOASSOCIATED VIRUS, TAKE OUT 978 00:35:59,560 --> 00:36:01,920 VIRAL DNA AND PUT IN THERAPEUTIC 979 00:36:01,920 --> 00:36:03,680 HUMAN DNAS AND MAKE DIFFERENT 980 00:36:03,680 --> 00:36:06,040 TREATMENTS OUT OF THEM, THAT IS 981 00:36:06,040 --> 00:36:07,360 THE SENSE WE MEAN BY GENE 982 00:36:07,360 --> 00:36:07,560 THERAPY. 983 00:36:07,560 --> 00:36:09,840 YOU CAN THINK OF THESE AAV 984 00:36:09,840 --> 00:36:12,560 VECTORS AS DELIVERY BOXES THAT 985 00:36:12,560 --> 00:36:14,920 ARE PRE-ADDRESSED IF YOU WILL TO 986 00:36:14,920 --> 00:36:16,000 DELIVER THERAPEUTIC GENES TO 987 00:36:16,000 --> 00:36:17,600 TARGET TISSUES. 988 00:36:17,600 --> 00:36:19,400 THEY CAN BE SINGLE TISSUES OR 989 00:36:19,400 --> 00:36:22,120 MULTIPLE TISSUES SO BUN WE CHOSE 990 00:36:22,120 --> 00:36:25,880 FOR PAGT CALLED AAV 9 THAT 991 00:36:25,880 --> 00:36:27,160 DELIVERS GENES MUSCLE AND LIVER 992 00:36:27,160 --> 00:36:28,680 AS WELL AS OTHER TISSUES. 993 00:36:28,680 --> 00:36:30,680 SO WE CAN USE THAT SAME DELIVERY 994 00:36:30,680 --> 00:36:32,320 BOX IF YOU WILL TO TREAT 995 00:36:32,320 --> 00:36:34,200 DISEASES THAT AFFECT BOTH MUSCLE 996 00:36:34,200 --> 00:36:38,640 AND LIVER WITHIN THE PEGT 997 00:36:38,640 --> 00:36:39,360 PROGRAM. 998 00:36:39,360 --> 00:36:40,040 NEXT SLIDE. 999 00:36:40,040 --> 00:36:41,880 SO TO SEE WHAT WE ARE DOING HERE 1000 00:36:41,880 --> 00:36:44,760 YOU CAN CONTRAST TRADITIONAL 1001 00:36:44,760 --> 00:36:45,880 CLINICAL DEVELOPMENT PARADIGMS 1002 00:36:45,880 --> 00:36:47,640 SO IMAGINE GENE THERAPIES FOR 1003 00:36:47,640 --> 00:36:52,560 THESE FOUR DIFFERENT RARE 1004 00:36:52,560 --> 00:36:57,720 GENETIC DISORDERS, ORGANIC 1005 00:36:57,720 --> 00:36:58,960 ACIDEMIAS DEFICIENCY AND 1006 00:36:58,960 --> 00:37:02,160 NEUROMUSCULAR JUNCTION DISORDERS 1007 00:37:02,160 --> 00:37:05,920 DOCKS 7 AND COQ DEFICIENCY. 1008 00:37:05,920 --> 00:37:11,560 SO THE TRADITIONAL IS GO THROUGH 1009 00:37:11,560 --> 00:37:13,400 DISEASES BOTTOM TO TOP IN 1010 00:37:13,400 --> 00:37:15,120 PARALLEL DOING EVERYTHING SAME 1011 00:37:15,120 --> 00:37:16,640 DESPITE YOU ARE USING THE SAME 1012 00:37:16,640 --> 00:37:21,160 DELIVERY SYSTEM, THE SAME 1013 00:37:21,160 --> 00:37:22,640 FACILITY FOR ALL FOUR DISEASE. 1014 00:37:22,640 --> 00:37:25,800 IF YOU LOOK AT IT QUITE 1015 00:37:25,800 --> 00:37:28,840 DUPLICATIVE, WE HOPE TO DO IN 1016 00:37:28,840 --> 00:37:31,000 PAGT IS ESSENTIALLY AS WE GO TO 1017 00:37:31,000 --> 00:37:33,240 THE FDA SAY LOOK, WE HAVE THE 1018 00:37:33,240 --> 00:37:36,200 SAME PLATFORM VECTOR, THE SAME 1019 00:37:36,200 --> 00:37:39,400 AAV 9, MADE MT. SAME FACILITY, 1020 00:37:39,400 --> 00:37:42,280 CAN WE REDO SOME OF THESE STEPS 1021 00:37:42,280 --> 00:37:45,480 PERHAPS EVEN SKIP THESE AS WE GO 1022 00:37:45,480 --> 00:37:46,240 INTO START UP THESE CLINICAL 1023 00:37:46,240 --> 00:37:46,760 TRIALS. 1024 00:37:46,760 --> 00:37:48,600 SO THINGS LIKE FOR EXAMPLE THE 1025 00:37:48,600 --> 00:37:50,320 BIODISTRIBUTION OF THE DRUG. 1026 00:37:50,320 --> 00:37:51,920 YOU WOULD IMAGINE WOULDN'T HAVE 1027 00:37:51,920 --> 00:37:54,680 TO CHANGE BECAUSE IT IS BASED ON 1028 00:37:54,680 --> 00:37:56,200 AAV 9 VECTOR AND TO BE 1029 00:37:56,200 --> 00:37:57,160 INDEPENDENT OF THE GENE THAT IS 1030 00:37:57,160 --> 00:37:58,280 IN IT. 1031 00:37:58,280 --> 00:37:59,960 SO OUR IDEA HERE IS AS WE MOVE 1032 00:37:59,960 --> 00:38:04,880 FORWARD WITH THESE DISCUSSANTS 1033 00:38:04,880 --> 00:38:08,960 OF THE FDA APPROVED IMD AS WE 1034 00:38:08,960 --> 00:38:10,760 ASK QUESTIONS PROPOSE WAYS TO 1035 00:38:10,760 --> 00:38:11,960 STREAMLINE THE PROCESS. 1036 00:38:11,960 --> 00:38:13,480 AS HAY RESPOND TO US WE WILL 1037 00:38:13,480 --> 00:38:14,880 MAKE IT AVAILABLE TO OTHERS SO 1038 00:38:14,880 --> 00:38:15,800 EVERYBODY IN THE COMMUNITY CAN 1039 00:38:15,800 --> 00:38:16,520 LEARN WITH US. 1040 00:38:16,520 --> 00:38:18,680 WE CAN REALLY SEE HOW MUCH WE 1041 00:38:18,680 --> 00:38:20,600 CAN EXPLOIT THE PLATFORM 1042 00:38:20,600 --> 00:38:22,240 CAPACITY OF AAVs FOR HUMAN 1043 00:38:22,240 --> 00:38:25,560 GENE THERAPY AN RARE DISEASES. 1044 00:38:25,560 --> 00:38:26,960 NEXT SLIDE. 1045 00:38:26,960 --> 00:38:31,320 SO THE STATUS OF PAGT VERSUS 1046 00:38:31,320 --> 00:38:34,000 FAIL EARLY STAGE WE HAVE A LEAD 1047 00:38:34,000 --> 00:38:39,840 CANDIDATE FOR ONE DISEASE, PCCA 1048 00:38:39,840 --> 00:38:40,960 DEFICIENCY, METABOLIC DISEASE 1049 00:38:40,960 --> 00:38:43,000 STUDIED MANY YEARS BY CHUCK, ONE 1050 00:38:43,000 --> 00:38:46,280 OF OUR COLLABORATORS. 1051 00:38:46,280 --> 00:38:48,760 AS WAS MENTIONED WE OBTAINED AN 1052 00:38:48,760 --> 00:38:51,560 ORPHAN DESIGNATION IN OCTOBER OF 1053 00:38:51,560 --> 00:38:54,560 2021 AND ANNE PARISER WORKED ON 1054 00:38:54,560 --> 00:38:56,000 THAT EFFORT WE ARE WORKING ON A 1055 00:38:56,000 --> 00:38:57,360 WHITE PAPER TO DESCRIBE THAT AND 1056 00:38:57,360 --> 00:38:59,040 MAKE THE DOCUMENTS PUBLIC SO 1057 00:38:59,040 --> 00:38:59,960 OTHERS BENEFIT FROM THEM. 1058 00:38:59,960 --> 00:39:01,760 WE ALSO HAD AN INTERACT MEETING 1059 00:39:01,760 --> 00:39:04,360 WITH THE FDA CBER IN JULY AND 1060 00:39:04,360 --> 00:39:06,280 PLAN TO WRITE A PAPER MAKE THESE 1061 00:39:06,280 --> 00:39:08,840 DOCUMENTS PUBLIC. 1062 00:39:08,840 --> 00:39:10,640 ULTIMATELY ALSO RARE PEDIATRIC 1063 00:39:10,640 --> 00:39:13,440 DISEASE DESIGNATION APPLICATION. 1064 00:39:13,440 --> 00:39:15,040 AND AGAIN CONTINUE TO GO THROUGH 1065 00:39:15,040 --> 00:39:16,520 THE PROCESS, MAKING THE 1066 00:39:16,520 --> 00:39:18,000 INFORMATION PUBLIC SO OTHERS CAN 1067 00:39:18,000 --> 00:39:25,480 LEARN AND BENEFIT FROM IT. 1068 00:39:25,480 --> 00:39:27,720 NOW MOVING TO ANOTHER EFFORT WE 1069 00:39:27,720 --> 00:39:32,120 SPOKE GENE THERAPY CONSORTIUM OF 1070 00:39:32,120 --> 00:39:33,560 BGTC WHICH ALSO WAS LAST 1071 00:39:33,560 --> 00:39:34,560 COUNCIL, TO SOME EXTENT THE IDEA 1072 00:39:34,560 --> 00:39:36,800 IS THE SAME, IT IS TO IDENTIFY 1073 00:39:36,800 --> 00:39:38,240 AND STREAMLINE THE PROCESS OF 1074 00:39:38,240 --> 00:39:41,200 STARTING UP AAV GENE THERAPY 1075 00:39:41,200 --> 00:39:42,360 TRIALS BUT SIGNIFICANT 1076 00:39:42,360 --> 00:39:43,640 DIFFERENCE HERE IS THIS IS A 1077 00:39:43,640 --> 00:39:47,440 PUBLIC PRIVATE PARTNERSHIP SO IT 1078 00:39:47,440 --> 00:39:49,000 INVOLVES -- ORGANIZED BY THE 1079 00:39:49,000 --> 00:39:52,080 FOUNDATION FOR NIH. 1080 00:39:52,080 --> 00:39:54,760 WHICH IS NOT THE NIH SO WE HAVE 1081 00:39:54,760 --> 00:39:56,560 DIFFERENT OPPORTUNITIES THERE IN 1082 00:39:56,560 --> 00:39:58,640 PARTICULAR OPPORTUNITIES TO WORK 1083 00:39:58,640 --> 00:40:00,760 WITH DIFFERENT BIOTECH COMPANIES 1084 00:40:00,760 --> 00:40:05,040 AND RELATING THIS WITH PFIZER 1085 00:40:05,040 --> 00:40:07,120 AND VERY IMPORTANTLY PETER MARKS 1086 00:40:07,120 --> 00:40:09,040 DIRECTOR OF CENTER FOR 1087 00:40:09,040 --> 00:40:10,880 BIOLODGEICS AT FDA. 1088 00:40:10,880 --> 00:40:13,200 NEXT SLIDE. 1089 00:40:13,200 --> 00:40:14,760 WE HAVE A LARGE NUMBER OF 1090 00:40:14,760 --> 00:40:16,880 PARTNERS IN THIS PARTICULAR 1091 00:40:16,880 --> 00:40:18,160 ACCELERATED MEDICINES PROGRAM, I 1092 00:40:18,160 --> 00:40:20,280 THINK THE IS A LARGEST ONE WE 1093 00:40:20,280 --> 00:40:21,640 HAVE MANY DIFFERENT NIH 1094 00:40:21,640 --> 00:40:24,160 INSTITUTES AND CENTERS ON THE 1095 00:40:24,160 --> 00:40:25,760 LEFT, PRIVATE SECTORS ON THE 1096 00:40:25,760 --> 00:40:26,200 RIGHT. 1097 00:40:26,200 --> 00:40:30,760 AND IN FACT WE CONTINUE TO ADD 1098 00:40:30,760 --> 00:40:32,160 AS THE PROGRAM LAWRNLGS WE HAVE 1099 00:40:32,160 --> 00:40:34,560 INCREASING INTEREST AND BEING 1100 00:40:34,560 --> 00:40:36,880 CONTACTED FOR MORE PARTNERSHIPS 1101 00:40:36,880 --> 00:40:40,440 WHICH IS WELCOME. 1102 00:40:40,440 --> 00:40:43,960 SO THERE IS TWO COMPONENTS OF 1103 00:40:43,960 --> 00:40:47,640 BTTC, BASIC SCIENCE COMPONENT 1104 00:40:47,640 --> 00:40:50,160 FOCUSED ON BETTER UNDERSTANDING 1105 00:40:50,160 --> 00:40:51,760 OPTIMIZING IF YOU WILL THE BASIC 1106 00:40:51,760 --> 00:40:54,520 BIOLOGY OF AAV AS IT IS RELEVANT 1107 00:40:54,520 --> 00:40:58,400 TO MAKING AAV VECTORS FOR HUMAN 1108 00:40:58,400 --> 00:41:00,360 GENE THERAPY AND ENHANCING 1109 00:41:00,360 --> 00:41:02,160 THERAPEUTIC GENE EXPRESSION ONCE 1110 00:41:02,160 --> 00:41:04,360 IT GETS TO PATIENTS. 1111 00:41:04,360 --> 00:41:08,560 PART WILL INVOLVE HIGH THROUGH 1112 00:41:08,560 --> 00:41:10,000 PUT ASSAYS THAT WILL BE CARRIED 1113 00:41:10,000 --> 00:41:11,560 OUT BY THE NCATS INTRAMURAL 1114 00:41:11,560 --> 00:41:13,280 PROGRAM. 1115 00:41:13,280 --> 00:41:16,040 AND THEN THE LARGER PART OF THE 1116 00:41:16,040 --> 00:41:18,040 PROGRAM IS CLINICAL COMPONENT 1117 00:41:18,040 --> 00:41:20,680 WHERE TRYING TO ADVANCE AAV 1118 00:41:20,680 --> 00:41:23,160 THERAPIES FOR THE SPOKE CLINICAL 1119 00:41:23,160 --> 00:41:24,560 APPLICATIONS, TRYING TO 1120 00:41:24,560 --> 00:41:26,560 HARMONIZE BEST PRACTICES FOR 1121 00:41:26,560 --> 00:41:28,200 MANUFACTURING AND ASSAY 1122 00:41:28,200 --> 00:41:32,200 THERAPEUTICS. 1123 00:41:32,200 --> 00:41:33,320 IDENTIFIES STREAMLINE REGULATORY 1124 00:41:33,320 --> 00:41:33,760 PATHWAY. 1125 00:41:33,760 --> 00:41:37,200 THE FOCUS OF THIS THE BESPOKE 1126 00:41:37,200 --> 00:41:39,200 BECAUSE FOCUSING HERE ON DISEASE 1127 00:41:39,200 --> 00:41:40,760 OF NO COMMERCIAL INTEREST BY 1128 00:41:40,760 --> 00:41:44,360 VIRTUE OF FACT THEY ARE LOW 1129 00:41:44,360 --> 00:41:46,520 PREVALENCE THIS IS HOW THIS 1130 00:41:46,520 --> 00:41:47,640 PROGRAM EXIST IN PRE-CLINICAL 1131 00:41:47,640 --> 00:41:50,080 SPACE WHICH IS A REQUIREMENT FOR 1132 00:41:50,080 --> 00:41:52,400 THESE AMP PROGRAMS. 1133 00:41:52,400 --> 00:41:55,680 NEXT SLIDE. 1134 00:41:55,680 --> 00:41:57,000 BECAUSE JONI MENTIONED THIS, I 1135 00:41:57,000 --> 00:41:58,840 DON'T WANT TO TAKE TOO MUCH TIME 1136 00:41:58,840 --> 00:42:01,600 BUT KEY QUESTION IS HOW TO 1137 00:42:01,600 --> 00:42:03,160 IDENTIFY DISEASE THAT WILL BE 1138 00:42:03,160 --> 00:42:05,160 INCLUDED IN THE BGGT CLINICAL 1139 00:42:05,160 --> 00:42:06,400 COMPONENT SO WE ARE NOT PROCESS 1140 00:42:06,400 --> 00:42:08,760 OF FIGURING OUT THAT NOW. 1141 00:42:08,760 --> 00:42:10,320 THIS IS VERY IMPORTANT FOR 1142 00:42:10,320 --> 00:42:11,760 PEOPLE LISTENING, WE HAVE AN 1143 00:42:11,760 --> 00:42:14,160 OPEN PROCESS FOR PEOPLE NOMINATE 1144 00:42:14,160 --> 00:42:18,280 DISEASE FOR PARTICIPATION IN THE 1145 00:42:18,280 --> 00:42:19,720 BGTC, THAT PROCESS CLOSES 1146 00:42:19,720 --> 00:42:21,480 FEBRUARY 18, FOR THOSE WHO ARE 1147 00:42:21,480 --> 00:42:23,480 INTERESTED THERE IS A LINK 1148 00:42:23,480 --> 00:42:26,200 THERE, YOU CAN FIND THE 1149 00:42:26,200 --> 00:42:27,920 INFORMATION ON THE F NIH 1150 00:42:27,920 --> 00:42:28,520 WEBSITE. 1151 00:42:28,520 --> 00:42:31,200 WE ALSO HAD A WEBINAR COUPLE OF 1152 00:42:31,200 --> 00:42:32,600 WEEKS AGO, YOU CAN FIND THAT 1153 00:42:32,600 --> 00:42:33,760 WEBSITE AS WELL AND OF COURSE IF 1154 00:42:33,760 --> 00:42:35,240 YOU NEED ADDITIONAL INFORMATION 1155 00:42:35,240 --> 00:42:37,680 YOU CAN CONTACT US. 1156 00:42:37,680 --> 00:42:41,600 NEXT SLIDE. 1157 00:42:41,600 --> 00:42:44,360 THINK ABOUT MONOGENIC DISEASE, 1158 00:42:44,360 --> 00:42:47,120 PERHAPS THE BROADEST THERAPEUTIC 1159 00:42:47,120 --> 00:42:48,160 PLATFORM OF ALL HA WE CAN 1160 00:42:48,160 --> 00:42:49,400 IMAGINE WOULD BE GENOME EDITING 1161 00:42:49,400 --> 00:42:51,640 WHERE YOU CAN ACTUALLY NOW 1162 00:42:51,640 --> 00:42:54,480 ACTUALLY POSSIBLE TO GO IN AND 1163 00:42:54,480 --> 00:42:56,280 DESIGN ENZYMES THAT WILL GO AND 1164 00:42:56,280 --> 00:42:57,840 CORRECT THE GENETIC 1165 00:42:57,840 --> 00:42:59,280 ABNORMALITIES LEADING TO OR 1166 00:42:59,280 --> 00:43:00,960 UNDERLYING MONOGENIC DISEASE. 1167 00:43:00,960 --> 00:43:03,520 AND SO THIS PROGRAM IS ONE THAT 1168 00:43:03,520 --> 00:43:05,720 IS GOING FOR A WHILE UNDER THE 1169 00:43:05,720 --> 00:43:08,480 NIH COMMON FUND, THE SOMATIC 1170 00:43:08,480 --> 00:43:10,360 CELL EDITING PROGRAM WHERE GOALS 1171 00:43:10,360 --> 00:43:12,240 ARE TO LOWER BARRIERS TO NEW 1172 00:43:12,240 --> 00:43:15,640 GENOME EDITING THERAPIES BY 1173 00:43:15,640 --> 00:43:18,960 ANIMAL MODELS FOR TESTING GENE, 1174 00:43:18,960 --> 00:43:20,640 ASSESSING UNINTENDED BIOLOGICAL 1175 00:43:20,640 --> 00:43:22,160 EFFECTS OF GENOME EDITING. 1176 00:43:22,160 --> 00:43:26,480 BIG COMPONENT ON IMPROVING 1177 00:43:26,480 --> 00:43:27,600 DELIVERING MACHINERY, EXPANDING 1178 00:43:27,600 --> 00:43:29,320 THE REPERTOIRE AND WE HAD 1179 00:43:29,320 --> 00:43:30,320 DISSEMINATION AND COORDINATION 1180 00:43:30,320 --> 00:43:32,360 SYSTEM THAT WILL MAKE AVAILABLE 1181 00:43:32,360 --> 00:43:34,880 IN THE PUBLIC WHAT WE CALL TOOL 1182 00:43:34,880 --> 00:43:37,200 KIT HIGHLIGHTING ALL THE 1183 00:43:37,200 --> 00:43:40,400 TECHNOLOGIES DEVELOPED IN THE 1184 00:43:40,400 --> 00:43:43,200 SCGE PROGRAM. 1185 00:43:43,200 --> 00:43:44,040 PART OF THE REASON WE ARE 1186 00:43:44,040 --> 00:43:46,560 TALKING ABOUT IT HERE IS BECAUSE 1187 00:43:46,560 --> 00:43:50,080 OF OTHER PROGRAM FUNDED BY 1188 00:43:50,080 --> 00:43:52,760 COMMON FUND THE LEADERSHIP 1189 00:43:52,760 --> 00:43:54,280 INVOLVES NCATS WORKING WITH 1190 00:43:54,280 --> 00:43:55,320 HINDZ. 1191 00:43:55,320 --> 00:43:56,920 AND THIS SHOWS THE VARIOUS 1192 00:43:56,920 --> 00:43:58,760 NUMBERS OF WORKING GROUP, LIKE 1193 00:43:58,760 --> 00:43:59,960 MANY OTHER EFFORTS ARE VERY 1194 00:43:59,960 --> 00:44:04,080 COLLABORATIVE. 1195 00:44:04,080 --> 00:44:05,120 EFFORT INVOLVING PROGRAM 1196 00:44:05,120 --> 00:44:06,000 OFFICERS FROM DIFFERENT PARTS OF 1197 00:44:06,000 --> 00:44:10,200 NIH. 1198 00:44:10,200 --> 00:44:11,440 COMMON FUND PROGRAMS ARE FIVE 1199 00:44:11,440 --> 00:44:12,880 YEARS AT A TIME AND THERE'S 1200 00:44:12,880 --> 00:44:17,440 CHANCE FOR ONE ADDITIONAL STAGE 1201 00:44:17,440 --> 00:44:20,360 OF THE PROGRAM, A PHASE 2 FOR 1202 00:44:20,360 --> 00:44:21,880 ANOTHER FIVE YEARS SO WE DID IN 1203 00:44:21,880 --> 00:44:24,680 FACT PROPOSE TO DO A PHASE 2 OF 1204 00:44:24,680 --> 00:44:25,360 SEG PROGRAM. 1205 00:44:25,360 --> 00:44:27,640 WE HAD TO BRING THAT TO WHAT IS 1206 00:44:27,640 --> 00:44:29,360 CALLED THE COUNCIL OF COUNCILS 1207 00:44:29,360 --> 00:44:32,800 THAT APPROVES NEW PROGRAMS FROM 1208 00:44:32,800 --> 00:44:34,280 OFFICE OF NIH DIRECTOR AND THAT 1209 00:44:34,280 --> 00:44:36,040 WAS DONE IN SEPTEMBER OF THIS 1210 00:44:36,040 --> 00:44:39,280 YEAR AND ACCEPTED. 1211 00:44:39,280 --> 00:44:42,360 PHASE 2 OF THE SCEG PROGRAM 1212 00:44:42,360 --> 00:44:44,040 MOVES FARTHER TOWARDS THE RIGHT 1213 00:44:44,040 --> 00:44:46,760 IF YOU WILL TOWARDS CLINICAL 1214 00:44:46,760 --> 00:44:48,280 TRIALS, WE WILL HAVE PLANNING AT 1215 00:44:48,280 --> 00:44:49,720 LEAST FOUR DIFFERENT 1216 00:44:49,720 --> 00:44:54,760 INITIATIVES, ONE ON REGULATORY 1217 00:44:54,760 --> 00:44:57,760 READY ASSAY DEVELOPMENT, ONE ON 1218 00:44:57,760 --> 00:45:00,480 IND ENABLING STUDIES, 1219 00:45:00,480 --> 00:45:01,280 DISSEMINATION COORDINATING 1220 00:45:01,280 --> 00:45:04,160 CENTER BUT THE MAIN ONE IS 1221 00:45:04,160 --> 00:45:05,760 INITIATIVE THREE WHICH WILL 1222 00:45:05,760 --> 00:45:06,840 SUPPORT PLATFORM CLINICAL TRIALS 1223 00:45:06,840 --> 00:45:12,160 OF GENOME EDITORS. 1224 00:45:12,160 --> 00:45:16,120 SO THIS THIRD INITIATIVE IS 1225 00:45:16,120 --> 00:45:18,680 FOCUSED ON HOW TO DEMONSTRATE 1226 00:45:18,680 --> 00:45:19,680 EFFICIENT CLINICAL TRIAL 1227 00:45:19,680 --> 00:45:21,920 STRATEGY TO PERFORM PLATFORM 1228 00:45:21,920 --> 00:45:23,000 GENOME EDITING CLINICAL TRIALS. 1229 00:45:23,000 --> 00:45:25,440 AND THE GOAL HERE IS TO SUPPORT 1230 00:45:25,440 --> 00:45:31,040 GENOME EDITING TRIALS OF MORE 1231 00:45:31,040 --> 00:45:32,320 THAN ONE DISEASE AT A TIME, THE 1232 00:45:32,320 --> 00:45:34,040 IDEA IS TO USE THE SAME DELIVERY 1233 00:45:34,040 --> 00:45:39,360 VEHICLE, THE SAME GENOME EDITOR. 1234 00:45:39,360 --> 00:45:41,040 AND THEN THE TRIAL ITSELF THOUGH 1235 00:45:41,040 --> 00:45:42,840 WOULD HAVE TO INCLUDE MORE THAN 1236 00:45:42,840 --> 00:45:44,560 ONE DISEASE SO YOU WOULD HAVE TO 1237 00:45:44,560 --> 00:45:47,440 USE DIFFERENT GUIDE RNAs AND 1238 00:45:47,440 --> 00:45:48,560 HAVE DIFFERENT CLINICAL OUTCOMES 1239 00:45:48,560 --> 00:45:50,320 AND HERE AGAIN THE QUESTION IS, 1240 00:45:50,320 --> 00:45:51,800 THE MAIN MILESTONES ARE HOW DO 1241 00:45:51,800 --> 00:45:53,760 YOU GET THAT REGULATORY 1242 00:45:53,760 --> 00:45:54,240 APPROVAL. 1243 00:45:54,240 --> 00:45:57,800 HOW DO YOU GET THE I INMD. 1244 00:45:57,800 --> 00:45:59,600 ONCE IND IS OBTAINED THE PROGRAM 1245 00:45:59,600 --> 00:46:01,000 WILL ALSO SUPPORT A SMALL 1246 00:46:01,000 --> 00:46:04,040 CLINICAL TRIAL OF THIS GENOME 1247 00:46:04,040 --> 00:46:05,040 EDITING CLINICAL TRIAL USING 1248 00:46:05,040 --> 00:46:06,160 THIS PLATFORM APPROACH. 1249 00:46:06,160 --> 00:46:12,240 TO REALLY NORMALIZE IN 1250 00:46:12,240 --> 00:46:14,160 DEVELOPMENT WHAT IS AN OBVIOUS 1251 00:46:14,160 --> 00:46:15,360 CLINICAL PLATFORM NOT DISEASE 1252 00:46:15,360 --> 00:46:19,160 SPECIFIC TREATMENT. 1253 00:46:19,160 --> 00:46:20,280 REQUIRED INTERACTIONS WITH THE 1254 00:46:20,280 --> 00:46:23,280 FDA TO INTERACT PRE-IND ET 1255 00:46:23,280 --> 00:46:28,200 CETERA AS I MENTIONED YESTERDAY. 1256 00:46:28,200 --> 00:46:31,280 THEN FINALLY WRAPPING UP MOVING 1257 00:46:31,280 --> 00:46:33,280 TOWARDS COLLABORATIVE ACTIVITIES 1258 00:46:33,280 --> 00:46:34,240 INCLUDING SCIENTIFIC MEANS, ET 1259 00:46:34,240 --> 00:46:34,640 CETERA. 1260 00:46:34,640 --> 00:46:38,640 NEXT SLIDE. 1261 00:46:38,640 --> 00:46:40,160 ONE OF THE SCIENTIFIC MEETINGS I 1262 00:46:40,160 --> 00:46:41,360 WANT TO HIGHLIGHT HERE IS A 1263 00:46:41,360 --> 00:46:43,320 WORKSHOP WE HELD IN 1264 00:46:43,320 --> 00:46:49,480 COLLABORATION PARTNERSHIP WITH 1265 00:46:49,480 --> 00:46:50,600 NICHD AND NINDS. 1266 00:46:50,600 --> 00:46:53,360 A SERIES OF MEETINGS HELD SUMMAR 1267 00:46:53,360 --> 00:46:56,080 OF THIS PAST YEAR. 1268 00:46:56,080 --> 00:46:58,200 IT HAS TO DO WITH THE 1269 00:46:58,200 --> 00:47:00,520 REALIZATION THESE GENE TARGETED 1270 00:47:00,520 --> 00:47:02,080 THERAPIES THAT WE HAVE BEEN 1271 00:47:02,080 --> 00:47:03,520 SAYING AFFECT MULTIPLE DISEASES 1272 00:47:03,520 --> 00:47:07,160 AT A TIME AND POTENTIALLY CAN 1273 00:47:07,160 --> 00:47:09,880 IMPACT LARGE NUMBER OF PATIENTS. 1274 00:47:09,880 --> 00:47:11,640 WITH MANY DIFFERENT GENETIC 1275 00:47:11,640 --> 00:47:12,320 DISEASES. 1276 00:47:12,320 --> 00:47:13,560 THE QUESTION IS HOW TO FIND ALL 1277 00:47:13,560 --> 00:47:15,240 THOSE PATIENTS HOW DO YOU 1278 00:47:15,240 --> 00:47:16,840 IDENTIFY ALL PATIENTS THAT MIGHT 1279 00:47:16,840 --> 00:47:18,080 BENEFIT FROM GENE TARGETED 1280 00:47:18,080 --> 00:47:20,640 THERAPIES. 1281 00:47:20,640 --> 00:47:22,600 GIVEN THAT WAY WE DIAGNOSE 1282 00:47:22,600 --> 00:47:25,760 PEOPLE IDENTIFY PATIENTS 1283 00:47:25,760 --> 00:47:28,560 INCLUDING THE WAY WE DO NEWBORN 1284 00:47:28,560 --> 00:47:29,760 SCREENING IS ONE DISEASE AT A 1285 00:47:29,760 --> 00:47:31,200 TIME BUT WE HAVE THERAPIES THAT 1286 00:47:31,200 --> 00:47:33,160 ARE THERAPEUTIC PLATFORMS 1287 00:47:33,160 --> 00:47:34,160 SOMETHING NEEDS TO CHANGE THERE 1288 00:47:34,160 --> 00:47:37,480 TO REALLY GET FULL THERAPEUTIC 1289 00:47:37,480 --> 00:47:38,880 BENEFIT OUT OF THESE NEW 1290 00:47:38,880 --> 00:47:39,240 APPROACH. 1291 00:47:39,240 --> 00:47:41,400 SO THIS WAS A WIDELY ATTENDED 1292 00:47:41,400 --> 00:47:46,640 SERIES OF WORKSHOPS 1500 VIRTUAL 1293 00:47:46,640 --> 00:47:47,840 ATTENDEES AND WORKING ON WHITE 1294 00:47:47,840 --> 00:47:50,360 PAPERS AND PUBLICATIONS TO 1295 00:47:50,360 --> 00:47:51,280 SUMMARIZE WHAT WE HAVE DONE AND 1296 00:47:51,280 --> 00:47:52,160 HOPEFULLY CONTINUE THIS 1297 00:47:52,160 --> 00:47:54,040 DISCUSSION. 1298 00:47:54,040 --> 00:47:57,560 A CLASSIC NCATS APPROACH PENDING 1299 00:47:57,560 --> 00:47:58,160 DIFFERENT STAKEHOLDERS. 1300 00:47:58,160 --> 00:48:00,960 NEXT SLIDE. 1301 00:48:00,960 --> 00:48:02,720 LOOKING TO THE FUTURE WE ARE 1302 00:48:02,720 --> 00:48:04,560 ALSO WORKING ON ANOTHER WORKSHOP 1303 00:48:04,560 --> 00:48:06,440 ON REGULATORY FITNESS IN RARE 1304 00:48:06,440 --> 00:48:08,360 DISEASE TRIALS, THAT RESPONSE 1305 00:48:08,360 --> 00:48:11,320 SAYING THE FDA CENTER FOR DRUGS, 1306 00:48:11,320 --> 00:48:13,760 SAY THE DATES THERE APRIL 7 AND 1307 00:48:13,760 --> 00:48:15,280 8 OF 2022. 1308 00:48:15,280 --> 00:48:18,160 THE REAL FOCUS IS TO HELP RARE 1309 00:48:18,160 --> 00:48:22,560 DISEASE INVESTIGATORS DEVELOP 1310 00:48:22,560 --> 00:48:26,360 THE BEST REGULATORY COMPATIBLE 1311 00:48:26,360 --> 00:48:27,560 INDs FOR THEIR CLINICAL TRIALS 1312 00:48:27,560 --> 00:48:29,080 AND RARE DISEASE SO THE INTENDED 1313 00:48:29,080 --> 00:48:30,720 AUDIENCE HERE REALLY ARE 1314 00:48:30,720 --> 00:48:34,960 ACADEMIC INVESTIGATORS AND SMALL 1315 00:48:34,960 --> 00:48:36,760 PHARMA BIOTECH COMPANIES 1316 00:48:36,760 --> 00:48:37,960 DEVELOPING DRUG FOR RARE DISEASE 1317 00:48:37,960 --> 00:48:41,040 AS WE ARE DEVELOPING THE PROGRAM 1318 00:48:41,040 --> 00:48:42,720 WILL HAVE HIGH CONCENTRATION OF 1319 00:48:42,720 --> 00:48:44,080 FDA SPEAKERS AND ORGANIZERS SO 1320 00:48:44,080 --> 00:48:48,200 WE WANT THIS TO BE A 1321 00:48:48,200 --> 00:48:49,040 COLLABORATIVE EFFORT THAT WOULD 1322 00:48:49,040 --> 00:48:50,240 BE OF BENEFIT TO BOTH OUR 1323 00:48:50,240 --> 00:48:51,960 INVESTIGATORS AND ULTIMATELY 1324 00:48:51,960 --> 00:48:53,720 ALSO TO THE -- OUR REGULATORY 1325 00:48:53,720 --> 00:49:00,000 COLLEAGUES. 1326 00:49:00,000 --> 00:49:01,920 ALSO OF COURSE AS JONI MENTIONED 1327 00:49:01,920 --> 00:49:04,360 GOT RARE DISEASE DAY COMING UP 1328 00:49:04,360 --> 00:49:06,000 FEBRUARY 28 OF THIS YEAR, 1329 00:49:06,000 --> 00:49:09,080 VIRTUAL OF COURSE GIVEN THE 1330 00:49:09,080 --> 00:49:10,560 PANDEMIC VARIOUS SESSIONS AS 1331 00:49:10,560 --> 00:49:12,000 LISTED THERE ON DIVERSITY AND 1332 00:49:12,000 --> 00:49:14,080 RARE DISEASE RESEARCH TOPIC OF 1333 00:49:14,080 --> 00:49:16,880 GREAT INTEREST, MARSHALL 1334 00:49:16,880 --> 00:49:21,840 MENTIONED RECENTLY, EXPANDING 1335 00:49:21,840 --> 00:49:22,800 OLIGONUCLEOTIDES, DIAGNOSTIC 1336 00:49:22,800 --> 00:49:24,720 ODYSSEY TELEHEALTH AND WE THINK 1337 00:49:24,720 --> 00:49:27,680 IT WILL BE A GREAT PROGRAM 1338 00:49:27,680 --> 00:49:28,720 WIDESPREAD REGISTRATION IS NOW 1339 00:49:28,720 --> 00:49:30,560 OPEN, CERTAINLY ENCOURAGE YOU TO 1340 00:49:30,560 --> 00:49:36,120 ATTEND THAT. 1341 00:49:36,120 --> 00:49:39,240 SO TO WRAP UP HERE THE TAKE HOME 1342 00:49:39,240 --> 00:49:41,000 MESSAGE AS ANNE MENTIONED FUTURE 1343 00:49:41,000 --> 00:49:43,000 IS WE GOT MANY RARE DISEASES AND 1344 00:49:43,000 --> 00:49:44,320 SOME OF THEM HAVE VERY SMALL 1345 00:49:44,320 --> 00:49:46,040 NUMBER OF PATIENTS AND FEW 1346 00:49:46,040 --> 00:49:47,360 TREATMENTS SO THE COLLECTIVELY 1347 00:49:47,360 --> 00:49:49,920 THIS IS A LARGE PUBLIC HEALTH 1348 00:49:49,920 --> 00:49:51,880 PROBLEM AND ONE THAT HAS MAJOR 1349 00:49:51,880 --> 00:49:53,560 ECONOMIC CONSEQUENCES TO OUR 1350 00:49:53,560 --> 00:49:55,000 PUBLIC -- OUR HEALTH SYSTEM IN 1351 00:49:55,000 --> 00:49:55,480 THE UNITED STATES. 1352 00:49:55,480 --> 00:49:57,760 AND I THINK THAT IS SOME OF THE 1353 00:49:57,760 --> 00:50:00,160 BIGGEST INSIGHTS MADE OVER THE 1354 00:50:00,160 --> 00:50:01,480 PAST -- LAST YEAR ACTUALLY. 1355 00:50:01,480 --> 00:50:04,760 WE ALSO HAVE SOME REALLY 1356 00:50:04,760 --> 00:50:05,360 UNPRECEDENTED SCIENTIFIC 1357 00:50:05,360 --> 00:50:06,400 OPPORTUNITIES SUCH AS GENOME 1358 00:50:06,400 --> 00:50:07,760 EDITING AND GENE THERAPY. 1359 00:50:07,760 --> 00:50:09,640 WHERE WE REALLY HAVE THE 1360 00:50:09,640 --> 00:50:10,680 SCIENTIFIC UNDERSTANDING TO 1361 00:50:10,680 --> 00:50:14,280 TREAT A LOT OF VERY SEVERE 1362 00:50:14,280 --> 00:50:16,600 SINGLE GENE DISORDERS BUT THE 1363 00:50:16,600 --> 00:50:18,600 LIMITING FACTOR IS NOT SO MUCH 1364 00:50:18,600 --> 00:50:21,000 THE SCIENCE BUT OPERATIONAL 1365 00:50:21,000 --> 00:50:22,760 LOGISTICAL BARRIERS, AND THAT IS 1366 00:50:22,760 --> 00:50:24,960 WHAT WE ARE TRYING TO APPROACH 1367 00:50:24,960 --> 00:50:26,520 AND WE WANT TO ENCOURAGE PEOPLE 1368 00:50:26,520 --> 00:50:28,720 TO THINK DIFFERENTLY AND ADAPT 1369 00:50:28,720 --> 00:50:31,360 ADOPT NEW STRATEGIES SUCH AS 1370 00:50:31,360 --> 00:50:32,920 MANY DISEASE AT A TIME, THINKING 1371 00:50:32,920 --> 00:50:34,920 HOW WE DEFINE DISEASE TO DESIGN 1372 00:50:34,920 --> 00:50:36,600 CLINICAL TRIALS AND PILOT 1373 00:50:36,600 --> 00:50:38,200 NON-TRADITIONAL APPROACH SO 1374 00:50:38,200 --> 00:50:44,840 OTHERS CAN PICK THEM UP. 1375 00:50:44,840 --> 00:50:47,520 I THINK THAT IS IT. 1376 00:50:47,520 --> 00:50:48,400 HAPPY TO ANSWER QUESTIONS. 1377 00:50:48,400 --> 00:50:48,560 T 1378 00:50:48,560 --> 00:50:50,680 >> THANK YOU PJ AND ANNE, REALLY 1379 00:50:50,680 --> 00:50:51,040 TERRIFIC. 1380 00:50:51,040 --> 00:50:53,360 ALSO UNDERSCORES THE IDEA THAT 1381 00:50:53,360 --> 00:50:55,040 THE THREE GOALS THAT WE TALK 1382 00:50:55,040 --> 00:50:56,800 ABOUT YESTERDAY, THESE SORTS OF 1383 00:50:56,800 --> 00:50:57,760 APPROACH ARE REALLY RIGHT IN 1384 00:50:57,760 --> 00:50:58,400 LINE WITH THOSE. 1385 00:50:58,400 --> 00:51:00,520 SO I WANT TO OPEN IT UP FOR 1386 00:51:00,520 --> 00:51:02,320 DISCUSSION FOR OUR COUNCIL. 1387 00:51:02,320 --> 00:51:04,040 I WILL MARSHALL I SEE YOUR HAND 1388 00:51:04,040 --> 00:51:08,800 UP FIRST SO GO AHEAD. 1389 00:51:08,800 --> 00:51:11,560 >> GREAT PRESENTATIONS, ANNE, 1390 00:51:11,560 --> 00:51:13,360 PJ, ANNE WE WILL MISS YOU, PJ I 1391 00:51:13,360 --> 00:51:14,760 HAVE KNOWN YOU A COUPLE OF YEARS 1392 00:51:14,760 --> 00:51:15,840 AND LOOKING FORWARD TO WORKING 1393 00:51:15,840 --> 00:51:16,640 WITH YOU. 1394 00:51:16,640 --> 00:51:18,840 IT LOOKS LIKE YOU ARE DOING 1395 00:51:18,840 --> 00:51:20,760 PRETTY GOOD COORDINATION WITH 1396 00:51:20,760 --> 00:51:21,840 OTHER GROUPS AND AS YOU KNOW 1397 00:51:21,840 --> 00:51:24,960 WITH A LOT OF THINGS LIKE 1398 00:51:24,960 --> 00:51:26,360 TELEHEALTH FDA TRIALS THESE 1399 00:51:26,360 --> 00:51:27,360 THINGS THERE ARE A LOT OF 1400 00:51:27,360 --> 00:51:29,720 EFFORTS GOING ON ODYSSEY AS WELL 1401 00:51:29,720 --> 00:51:32,320 TO LOOKING AHEAD WHERE DO YOU 1402 00:51:32,320 --> 00:51:36,280 SEE AREAS WE NEED TO BUILD FIRST 1403 00:51:36,280 --> 00:51:38,320 TO NOT DUPLICATE BUT SECOND PULL 1404 00:51:38,320 --> 00:51:39,840 THE COMMUNITY ALONG WITH NIH 1405 00:51:39,840 --> 00:51:41,320 LEADERSHIP; WHAT ARE YOUR BIG 1406 00:51:41,320 --> 00:51:49,360 OPPORTUNITIES YOU SEE COMING UP 1407 00:51:49,360 --> 00:51:53,280 >> I THINK WE HAVE BROKEN GROUND 1408 00:51:53,280 --> 00:51:54,880 IN TRYING TO START PILOTS IN A 1409 00:51:54,880 --> 00:51:59,080 LOT OF AREAS. 1410 00:51:59,080 --> 00:52:00,680 SO THERE'S ROOM FOR GROWTH IN 1411 00:52:00,680 --> 00:52:02,560 ALL OF THOSE. 1412 00:52:02,560 --> 00:52:05,760 I THINK THERE IS MOST DISEASE 1413 00:52:05,760 --> 00:52:07,320 THERE IS VERY LITTLE RESEARCH 1414 00:52:07,320 --> 00:52:09,400 GOING ON, CERTAINLY NOT A LOT OF 1415 00:52:09,400 --> 00:52:10,880 DUPLICATION. 1416 00:52:10,880 --> 00:52:14,480 SO WHAT WE LIKE TO SEE IS THE 1417 00:52:14,480 --> 00:52:16,800 ABILITY TO TAKE ON MORE 1418 00:52:16,800 --> 00:52:18,360 CLUSTERS, SOME WHICH PJ WAS 1419 00:52:18,360 --> 00:52:20,240 TALKING ABOUT WITH THESE MANY 1420 00:52:20,240 --> 00:52:22,800 DISEASES AT A TIME APPROACH. 1421 00:52:22,800 --> 00:52:24,880 I THINK THAT FROM THE 1422 00:52:24,880 --> 00:52:28,880 THERAPEUTICS AREA WE CAN SEAL 1423 00:52:28,880 --> 00:52:30,000 THAT ONLINE. 1424 00:52:30,000 --> 00:52:31,560 WHAT THERE IS NOT GOING ON RIGHT 1425 00:52:31,560 --> 00:52:33,040 NOW IS DIAGNOSTICS SO THERE'S 1426 00:52:33,040 --> 00:52:36,200 BEEN A LOT OF ATTENTION ON THE 1427 00:52:36,200 --> 00:52:37,560 NEWBORN AREA WHICH IS FANTASTIC 1428 00:52:37,560 --> 00:52:40,880 AND THERE IS PROGRESS THERE 1429 00:52:40,880 --> 00:52:43,400 ININCLUDING STEVEN KING AND 1430 00:52:43,400 --> 00:52:44,560 OTHER PEOPLE BUT WHAT ABOUT 1431 00:52:44,560 --> 00:52:46,960 OLDER PEOPLE L NOT IDENTIFIES BY 1432 00:52:46,960 --> 00:52:48,760 OLDER WE ARE TALKING TWO TO 1433 00:52:48,760 --> 00:52:50,760 FIVE-YEAR-OLD SO NOT OLDER BUT 1434 00:52:50,760 --> 00:52:53,160 THERE HASN'T BEEN ATTENTION 1435 00:52:53,160 --> 00:52:54,760 THERE SO WE ARE TAKING MORE INTO 1436 00:52:54,760 --> 00:52:55,320 THAT. 1437 00:52:55,320 --> 00:52:56,680 BUT I DON'T THINK THE NEEDLE HAS 1438 00:52:56,680 --> 00:53:02,160 MOVED THERE QUITE AS MUCH. 1439 00:53:02,160 --> 00:53:06,680 SO HOPING TO CALL MORE 1440 00:53:06,680 --> 00:53:07,280 ATTENTION. 1441 00:53:07,280 --> 00:53:10,640 >> ARE YOU -- ON DIEG NOIS 1442 00:53:10,640 --> 00:53:12,400 NOSSICS AND -- DIEG NOICS AND 1443 00:53:12,400 --> 00:53:12,840 RARE DISEASE? 1444 00:53:12,840 --> 00:53:15,840 THAT'S A A COLLABORATIVE GROUP 1445 00:53:15,840 --> 00:53:17,720 THAT HAS LOTS GOING ON 1446 00:53:17,720 --> 00:53:19,880 >> WE HAVEN'T YET BUT THAT IS A 1447 00:53:19,880 --> 00:53:23,400 REALLY GOOD IDEA PARTICULARLY AS 1448 00:53:23,400 --> 00:53:27,800 WE START TO GET PROJECTS FUNDED 1449 00:53:27,800 --> 00:53:31,360 AND WE HAVE RESULTS TO SHOW AND 1450 00:53:31,360 --> 00:53:32,240 SHARE THAT WOULD BE A GOOD 1451 00:53:32,240 --> 00:53:34,800 OPPORTUNITY. 1452 00:53:34,800 --> 00:53:36,360 >> MAYBE FOLLOW-UP ON MARSHALL'S 1453 00:53:36,360 --> 00:53:36,960 QUESTION. 1454 00:53:36,960 --> 00:53:38,960 I THINK AS WE HAVE SEEN, WE 1455 00:53:38,960 --> 00:53:40,560 CERTAINLY HAVE ENGAGEMENT WITH 1456 00:53:40,560 --> 00:53:43,800 THE FDA BOTH CBER WHICH IS 1457 00:53:43,800 --> 00:53:47,360 REALLY WONDERFUL, ONE AREA I 1458 00:53:47,360 --> 00:53:48,480 THINK ABOUT THESE DAYS AND WE 1459 00:53:48,480 --> 00:53:50,280 HAVE HIGHLIGHTED THIS, IS THE 1460 00:53:50,280 --> 00:53:53,800 ISSUE OF DIVERSITY AND HOW WE 1461 00:53:53,800 --> 00:53:55,680 DEAL WITH DIVERSE POPULATIONS IN 1462 00:53:55,680 --> 00:53:57,640 RARE DISEASES AND CLINICAL 1463 00:53:57,640 --> 00:54:01,560 TRIALS GETTING THESE ADVANCES TO 1464 00:54:01,560 --> 00:54:05,640 AS MANY PEOPLE AS CAN POSSIBLY 1465 00:54:05,640 --> 00:54:08,400 BENEFIT FROM THEM. 1466 00:54:08,400 --> 00:54:10,240 >> MATTHIAS. 1467 00:54:10,240 --> 00:54:14,920 >> CONGRATULATIONS, MUST BE VERY 1468 00:54:14,920 --> 00:54:18,760 GRATIFYING TO SEE WHERE ORDR HAS 1469 00:54:18,760 --> 00:54:21,160 GONE WHICH CREATE EXCITING TIMES 1470 00:54:21,160 --> 00:54:22,760 YOU CAN HAND -- REALLY A 1471 00:54:22,760 --> 00:54:27,720 PLEASURE TO WORK WITH YOU AND PJ 1472 00:54:27,720 --> 00:54:30,240 HAPPY TO SEE Y'ALL LOOK LOOKING 1473 00:54:30,240 --> 00:54:33,440 FORWARD TO EXCITING TIMES, YOU 1474 00:54:33,440 --> 00:54:38,800 ARE LOOKING OUTSIDE THE NCATS, 1475 00:54:38,800 --> 00:54:45,920 WORKING WITH WITH F NIH, A LOT 1476 00:54:45,920 --> 00:54:48,560 OF THE MOST INNOVATIVE FORCES TO 1477 00:54:48,560 --> 00:54:51,440 COME BOTH FROM ACT DEEM Y AND 1478 00:54:51,440 --> 00:54:53,040 ENTITIES. 1479 00:54:53,040 --> 00:54:54,240 AND WOULD STRONGLY ENCOURAGE 1480 00:54:54,240 --> 00:54:56,560 THAT AND F NIH IS A VERY 1481 00:54:56,560 --> 00:55:03,480 EFFECTIVE MEK ANYMOREFFECTIVE MECHANISM AN D KUDOS TO 1482 00:55:03,480 --> 00:55:04,840 GO THERE, ENVIRONMENT THERE IS. 1483 00:55:04,840 --> 00:55:07,080 KEEP YOUR EYES OPEN AND I THINK 1484 00:55:07,080 --> 00:55:12,480 YOU DO BEYOND GENE EDITING AND 1485 00:55:12,480 --> 00:55:16,440 THERE IS GUIDANCE CONCERNING SI 1486 00:55:16,440 --> 00:55:17,440 RNAs, THERAPEUTICS ARE MOVING 1487 00:55:17,440 --> 00:55:19,680 TO THE CLINIC. 1488 00:55:19,680 --> 00:55:22,200 THIS IS AN INCREDIBLE TIME WHERE 1489 00:55:22,200 --> 00:55:28,120 TIME WINDOW FROM IDENTIFYING TO 1490 00:55:28,120 --> 00:55:29,680 THERAPEUTIC STRATEGIES CAN BE 1491 00:55:29,680 --> 00:55:31,880 CUT DOWN TO MONTHS NOW AND THERE 1492 00:55:31,880 --> 00:55:36,560 IS ONE OF THE KEY FEATURES WE 1493 00:55:36,560 --> 00:55:38,120 DISCUSS 25% GOAL, JONI PUT IN 1494 00:55:38,120 --> 00:55:43,800 FRONT OF US, IS ACTUALLY 1495 00:55:43,800 --> 00:55:44,920 POTENTIALLY REACHABLE AND WANT 1496 00:55:44,920 --> 00:55:46,920 TO ENFORCE THE CONCEPT IF YOU 1497 00:55:46,920 --> 00:55:48,440 CAN BUNDLE STRATEGIES IF THERE 1498 00:55:48,440 --> 00:55:50,920 ARE COMMON TRIGGERS YOU CAN PULL 1499 00:55:50,920 --> 00:55:53,760 TO REVERSE STABILIZE DETAIN 1500 00:55:53,760 --> 00:55:54,960 FUNCTION IT MIGHT BE ONE OF THE 1501 00:55:54,960 --> 00:55:56,760 AREAS, MANY OF THESE INNOVATIVE 1502 00:55:56,760 --> 00:55:58,760 TOOLS CAN CONTINUE TO DO AND 1503 00:55:58,760 --> 00:56:01,880 THEN VERY MUCH LOOKING FORWARD 1504 00:56:01,880 --> 00:56:05,080 LATER THIS AFTERNOON TO DISCUSS 1505 00:56:05,080 --> 00:56:05,920 STEPS GOING FORWARD. 1506 00:56:05,920 --> 00:56:08,480 CONGRATULATIONS TO BOTH OF YOU. 1507 00:56:08,480 --> 00:56:09,200 >> THANK YOU. 1508 00:56:09,200 --> 00:56:14,080 >> THANK YOU FOR THE CHAT LINK 1509 00:56:14,080 --> 00:56:15,760 TO FDA GUIDANCE DOCUMENTS, GREAT 1510 00:56:15,760 --> 00:56:22,760 TO HAVE. 1511 00:56:22,760 --> 00:56:24,160 >> THANKS TO YOU FOR YOUR 1512 00:56:24,160 --> 00:56:25,480 LEADERSHIP IN ALL THESE 1513 00:56:25,480 --> 00:56:26,160 DIFFERENT PROJECTS. 1514 00:56:26,160 --> 00:56:30,080 I ALSO WANT TO EXTEND THANKS TO 1515 00:56:30,080 --> 00:56:32,640 YOUR TEAM, THERE HAVE BEEN SOME 1516 00:56:32,640 --> 00:56:35,640 HEROIC EFFORTS ON PART OF YOUR 1517 00:56:35,640 --> 00:56:39,320 TEAM MEMBERS THAT YOU TALKED 1518 00:56:39,320 --> 00:56:41,560 ABOUT. 1519 00:56:41,560 --> 00:56:44,320 FIRSTHAND, ERIC SAID WHO 1520 00:56:44,320 --> 00:56:45,880 NAVIGATED DATA WITH REGARDS TO 1521 00:56:45,880 --> 00:56:48,320 BURDEN DATA AND ICD DATA THIS 1522 00:56:48,320 --> 00:56:50,520 YEAR AND TIINA URV WHO LED THAT 1523 00:56:50,520 --> 00:56:53,760 SUMMAR WORKSHOP SERIES. 1524 00:56:53,760 --> 00:56:55,640 WHICH WAS HERDING A LOT OF CATS 1525 00:56:55,640 --> 00:56:57,320 OVER MONTHS AND NOW STILL DOING 1526 00:56:57,320 --> 00:56:58,800 THE SAME THING AS PUBLICATION 1527 00:56:58,800 --> 00:57:00,280 COME OUT SO THANK YOU TO THE 1528 00:57:00,280 --> 00:57:02,360 ENTIRE ORDR TEAM WORKING ON 1529 00:57:02,360 --> 00:57:06,480 BEHALF OF (INAUDIBLE). 1530 00:57:06,480 --> 00:57:07,720 APPLYING RESOURCE AS BUT MOVE 1531 00:57:07,720 --> 00:57:10,000 ONE OF YOUR PROJECTS FORWARD AND 1532 00:57:10,000 --> 00:57:13,200 THEN QUESTION ABOUT ONE OF THE 1533 00:57:13,200 --> 00:57:15,080 OTHERS. 1534 00:57:15,080 --> 00:57:16,160 ONE IS WITH RESPECT TO THE 1535 00:57:16,160 --> 00:57:21,160 MEETING YOU ARE WORKING ON THERE 1536 00:57:21,160 --> 00:57:22,680 IS EFFORT IN THE COMMUNITIES 1537 00:57:22,680 --> 00:57:24,000 BEEN ENGAGED THE LAST YEAR AND 1538 00:57:24,000 --> 00:57:26,240 ANNE PARTICIPATED IN THIS, WHERE 1539 00:57:26,240 --> 00:57:27,920 WE WENT THROUGH COLLECTIVELY ALL 1540 00:57:27,920 --> 00:57:30,480 THE GUIDANCES AND FDA PUT OUT, 1541 00:57:30,480 --> 00:57:31,640 RELATED TO THE INTEGRATION OF 1542 00:57:31,640 --> 00:57:33,360 PATIENT EXPERIENCE AND PATIENT 1543 00:57:33,360 --> 00:57:34,280 EXPERIENCE DATA. 1544 00:57:34,280 --> 00:57:37,760 ACROSS THE AGENCY. 1545 00:57:37,760 --> 00:57:39,400 AND ASSESS ACROSS THE MEDICAL 1546 00:57:39,400 --> 00:57:41,960 PRODUCT DEVELOPMENT LIFE CYCLE. 1547 00:57:41,960 --> 00:57:43,600 AND WE HAVE NOW A GUIDE THAT 1548 00:57:43,600 --> 00:57:44,960 WILL BE COMING OUT NEXT WEEK. 1549 00:57:44,960 --> 00:57:47,000 WE MET WITH THE FDA TWICE 1550 00:57:47,000 --> 00:57:53,840 THROUGHOUT THAT PROCESS WITH THE 1551 00:57:53,840 --> 00:57:55,320 AGENCY RELATED TO THAT, SO A 1552 00:57:55,320 --> 00:57:57,760 NICE JUMPING OFF POINT HIKING 1553 00:57:57,760 --> 00:57:59,320 ABOUT PLANNING, AS WELL AS MANY 1554 00:57:59,320 --> 00:58:02,200 PARTICIPANTS IN THOSE ROUND 1555 00:58:02,200 --> 00:58:03,280 TABLE MAKE GOOD SPEAKER 1556 00:58:03,280 --> 00:58:04,960 SOMETHING TO THINK ABOUT AS YOU 1557 00:58:04,960 --> 00:58:08,840 ARE PLANNING FOR THAT MEETING. 1558 00:58:08,840 --> 00:58:15,160 THE ZEBRA TRIGGER IS I HEARD YOU 1559 00:58:15,160 --> 00:58:17,280 I LOVE THIS PROJECT AND I WILL 1560 00:58:17,280 --> 00:58:19,520 HELP CLOSE THE DIAGNOSTIC GAP WE 1561 00:58:19,520 --> 00:58:21,160 KNOW IN OUR BURDEN STUDY WE 1562 00:58:21,160 --> 00:58:23,600 FOUND IS ABOUT SIX YEARS, 1563 00:58:23,600 --> 00:58:25,960 INCLUDES VISITS TO 17 1564 00:58:25,960 --> 00:58:27,040 SPECIALISTS IN THOSE SIX YEARS 1565 00:58:27,040 --> 00:58:33,640 WHICH IS REALLY MIND BOGGLING 1566 00:58:33,640 --> 00:58:34,760 AND ALSO WHEN YOU THINK WHO IS 1567 00:58:34,760 --> 00:58:36,360 NOT REPRESENTED IN THAT DATA IS 1568 00:58:36,360 --> 00:58:37,200 REALLY STRAIGHT GAME. 1569 00:58:37,200 --> 00:58:39,880 MY QUESTION AS YOU THINK ABOUT 1570 00:58:39,880 --> 00:58:41,640 THAT EFFORT ONE QUESTION I HAD, 1571 00:58:41,640 --> 00:58:43,320 ARE YOU WORKING WITH THE CDC AND 1572 00:58:43,320 --> 00:58:48,160 OTHER AGENCIES, AND PROFESSIONAL 1573 00:58:48,160 --> 00:58:51,360 SOCIETIES LIKE AAP OR ACMG, 1574 00:58:51,360 --> 00:58:54,200 NURSE PRACTICE EVERYTHING, PA 1575 00:58:54,200 --> 00:58:57,280 SOCIETIES NHDC AS YOU THINK NEXT 1576 00:58:57,280 --> 00:58:58,960 STEPS, THAT SEEMS TO ME BASED 1577 00:58:58,960 --> 00:59:01,680 SOMETHING NCATS PULL SON SOR 1578 00:59:01,680 --> 00:59:04,440 SHAH AROUND -- CONSORTIA AROUND 1579 00:59:04,440 --> 00:59:05,880 TALKING ABOUT WHAT TO DO NEXT 1580 00:59:05,880 --> 00:59:07,240 WITH THE DATA THEY ARE 1581 00:59:07,240 --> 00:59:07,960 COLLECTING. 1582 00:59:07,960 --> 00:59:09,680 >> WE ARE NOT WORKING WITH THESE 1583 00:59:09,680 --> 00:59:10,160 GROUPS YET. 1584 00:59:10,160 --> 00:59:12,920 BUT I DO THINK THAT'S A GREAT 1585 00:59:12,920 --> 00:59:15,320 SUGGESTION AND MAYBE SIMILAR TO 1586 00:59:15,320 --> 00:59:20,160 WHAT MENTION WITH MARSHALL, WE 1587 00:59:20,160 --> 00:59:24,400 ARE JUST EM BASHING ON THESE 1588 00:59:24,400 --> 00:59:27,360 INCLUDING WORKING WITH ANOTHER 1589 00:59:27,360 --> 00:59:29,760 DATA ANALYTICS CONTRACTOR TO TRY 1590 00:59:29,760 --> 00:59:32,280 TO TEST OUT SOME OF THESE THINGS 1591 00:59:32,280 --> 00:59:38,880 WE HAVE IDENTIFIED AS WELL AS 1592 00:59:38,880 --> 00:59:40,360 FUNDING A FEW GRANTS TO TRY TO 1593 00:59:40,360 --> 00:59:45,960 DEVELOP THESE STRATEGIES AS WE 1594 00:59:45,960 --> 00:59:47,400 GET MORE DATA AROUND THIS AND 1595 00:59:47,400 --> 00:59:48,480 THINGS THAT ARE ACTIONABLE WOULD 1596 00:59:48,480 --> 00:59:52,040 BE A GOOD TIME TO GO TO THESE 1597 00:59:52,040 --> 00:59:53,760 GROUPS TO SEE IF WE CAN'T TEST 1598 00:59:53,760 --> 00:59:55,440 OUT IN DIFFERENT ENVIRONMENTS 1599 00:59:55,440 --> 00:59:56,360 AND GET FEEDBACK. 1600 00:59:56,360 --> 01:00:00,480 SO THOSE ARE GREAT SUGGESTIONS. 1601 01:00:00,480 --> 01:00:02,680 >> THANK YOU. 1602 01:00:02,680 --> 01:00:05,320 >> BECKY. 1603 01:00:05,320 --> 01:00:07,560 >> GOING TO TAKE THIS A LITTLE 1604 01:00:07,560 --> 01:00:10,840 DIFFERENT AREA. 1605 01:00:10,840 --> 01:00:13,080 ONE THING I DIDN'T SEE 1606 01:00:13,080 --> 01:00:16,120 HIGHLIGHTED AND CERTAINLY A 1607 01:00:16,120 --> 01:00:18,600 MAJOR SET OF CONCERNS FOR 1608 01:00:18,600 --> 01:00:21,600 INDIVIDUALS WHO LIVED WITH A 1609 01:00:21,600 --> 01:00:23,240 RARE DISEASE, PARTICULARLY RARE 1610 01:00:23,240 --> 01:00:27,640 DISEASES HAD SIGNIFICANT IMPACT 1611 01:00:27,640 --> 01:00:32,480 ON ABILITY FOCUS ON A HOST OF 1612 01:00:32,480 --> 01:00:34,800 OVER CO-MORBID CONDITIONS THAT 1613 01:00:34,800 --> 01:00:38,160 OCCUR OR CO-MORBID SYMPTOMS THAT 1614 01:00:38,160 --> 01:00:40,160 OCCUR WITH THIS IS REALLY A 1615 01:00:40,160 --> 01:00:45,840 FOCUS ON IMPLEMENTATION SCIENCE. 1616 01:00:45,840 --> 01:00:48,560 WHERE MOST OF THE FOCUS TO DATE 1617 01:00:48,560 --> 01:00:52,240 HAS BEEN MUCH MORE ON CHILDREN 1618 01:00:52,240 --> 01:00:55,280 BUT WHEN YOU LOOK LONGITUDINALLY 1619 01:00:55,280 --> 01:00:56,600 ACROSS LIFE COURSE WHAT YOU FIND 1620 01:00:56,600 --> 01:00:59,720 IS THAT THOSE INTEGRATED SYSTEMS 1621 01:00:59,720 --> 01:01:02,160 OF SUPPORT THAT OCCUR ONCE 1622 01:01:02,160 --> 01:01:04,160 SOMEONE FINALLY GETS DIAGNOSIS 1623 01:01:04,160 --> 01:01:07,560 ARE NOT SUSTAINED AS YOU MOVE TO 1624 01:01:07,560 --> 01:01:11,720 THE ADULT YEARS AND EVEN ELDERLY 1625 01:01:11,720 --> 01:01:13,520 YEARS AND LIFE SPAN MAY NOT BE 1626 01:01:13,520 --> 01:01:15,360 SIGNIFICANTLY IMPACTED SO JUST 1627 01:01:15,360 --> 01:01:16,600 ENCOURAGE AS YOU MOVE FORWARD TO 1628 01:01:16,600 --> 01:01:17,960 THINK ABOUT THAT WHOLE 1629 01:01:17,960 --> 01:01:20,360 TRANSLATIONAL SPECTRUM. 1630 01:01:20,360 --> 01:01:25,800 AND THAT IMPLEMENTATION HOW TO 1631 01:01:25,800 --> 01:01:27,200 SUPPORT ACTIVITIES WHAT RESEARCH 1632 01:01:27,200 --> 01:01:28,960 SHOULD WE DO TO UNDERSTAND THE 1633 01:01:28,960 --> 01:01:32,160 BEST WAY TO LIVE MOST 1634 01:01:32,160 --> 01:01:34,360 EFFECTIVELY WITH THE RARE 1635 01:01:34,360 --> 01:01:34,960 DISEASES. 1636 01:01:34,960 --> 01:02:03,160 THAT WON'T YET HAVE A -- 1637 01:02:03,160 --> 01:02:14,080 >> THANK YOU, KRISTINA. 1638 01:02:14,080 --> 01:02:15,960 MANY OF YOU KNOW THIS IS ONE OF 1639 01:02:15,960 --> 01:02:17,840 MY SOAP BOX ISSUES. 1640 01:02:17,840 --> 01:02:21,760 SO I KNOW THEY ARE VERY FAMILIAR 1641 01:02:21,760 --> 01:02:24,880 WITH THIS MY FAMILY WENT THROUGH 1642 01:02:24,880 --> 01:02:26,680 IS INSANE GIVEN I'M HERE ON THIS 1643 01:02:26,680 --> 01:02:29,600 COUNCIL WITH ALL OF YOU TWO 1644 01:02:29,600 --> 01:02:30,840 BLOCKS FROM THE NIH AND IT WAS 1645 01:02:30,840 --> 01:02:35,560 STILL A HUGE ODYSSEY FOR US TO 1646 01:02:35,560 --> 01:02:36,920 GET CHARLOTTE'S DIAGNOSIS. 1647 01:02:36,920 --> 01:02:41,120 I SPENT THE DOCUMENT IN THERE I 1648 01:02:41,120 --> 01:02:44,440 SPOKE ABOUT IT AT THE RESEARCH 1649 01:02:44,440 --> 01:02:44,880 AMERICA SUMMIT. 1650 01:02:44,880 --> 01:02:47,120 YOU CAN READ OR WATCH THE VIDEO. 1651 01:02:47,120 --> 01:02:48,360 WHAT IS CHALLENGING IS MAKING 1652 01:02:48,360 --> 01:02:50,360 SURE FOLKS ON CAPITOL HILL HAVE 1653 01:02:50,360 --> 01:02:52,920 THE RIGHT EDUCATION AND 1654 01:02:52,920 --> 01:02:53,920 UNDERSTANDING ABOUT WHAT THE 1655 01:02:53,920 --> 01:02:57,440 PROBLEM REALLY IS. 1656 01:02:57,440 --> 01:02:59,440 THIS SOMETHING ANNIE AND 1657 01:02:59,440 --> 01:03:00,320 MARSHALL AND MANY HAVE BEEN 1658 01:03:00,320 --> 01:03:01,920 WORKING ON YEARS NOTICE POINT. 1659 01:03:01,920 --> 01:03:04,400 AND I LOVE WHAT ANNIE WAS SAYING 1660 01:03:04,400 --> 01:03:08,680 ABOUT POTENTIALLY CONVENING 1661 01:03:08,680 --> 01:03:09,960 SMOFTS MEDICAL SOCIETIES LIKE -- 1662 01:03:09,960 --> 01:03:11,560 SOME OF THE MEDICAL SOCIETIES 1663 01:03:11,560 --> 01:03:13,360 WHICH IS WORKING ON EDUCATING 1664 01:03:13,360 --> 01:03:17,360 HERE, BUT RIGHT NOW Y'ALL 1665 01:03:17,360 --> 01:03:19,520 PROBABLY SAW THINGS HARD WORK ON 1666 01:03:19,520 --> 01:03:21,480 EVERY LIFE AND ASSISTANCE FUND 1667 01:03:21,480 --> 01:03:24,480 AND BUNCH OF O OTHER GROUPS IN 1668 01:03:24,480 --> 01:03:26,920 CURES 2.0 THERE IS LANGUAGE 1669 01:03:26,920 --> 01:03:28,320 THERE FOR COVERAGE OF GENOMIC 1670 01:03:28,320 --> 01:03:28,920 SEQUENCING. 1671 01:03:28,920 --> 01:03:31,240 IT WAS A THREE YEAR BATTLE, TO 1672 01:03:31,240 --> 01:03:33,240 ENSURE THAT THAT LANGUAGE WHICH 1673 01:03:33,240 --> 01:03:37,760 AGAIN IS HOUSE BILL, COVERS 1674 01:03:37,760 --> 01:03:41,000 WHOLE GENOME WHOLE EXOME AND 1675 01:03:41,000 --> 01:03:42,360 PANELS. 1676 01:03:42,360 --> 01:03:43,280 THE PRINCIPLE BEING CLINICS 1677 01:03:43,280 --> 01:03:45,440 SHOULD HAVE THE DISCRETION TO 1678 01:03:45,440 --> 01:03:46,960 ORDER WHATEVER TESTS THEY DEEM 1679 01:03:46,960 --> 01:03:48,400 FIT AND SHOULDN'T BE SPENDING 1680 01:03:48,400 --> 01:03:50,080 THEIR TIME SEEKING INSURANCE 1681 01:03:50,080 --> 01:03:54,840 COVERAGE FOR THOSE TESTS. 1682 01:03:54,840 --> 01:03:56,480 ON THE SENATE SIDE TR IS 1683 01:03:56,480 --> 01:03:57,760 EXCLUSIVE TO WHOLE GENOME 1684 01:03:57,760 --> 01:04:00,560 SEQUENCING AND I HEARD NHGRI 1685 01:04:00,560 --> 01:04:01,960 WILL BE PART OF THAT 1686 01:04:01,960 --> 01:04:03,360 CONGRESSIONAL BRIEFING PANEL. 1687 01:04:03,360 --> 01:04:05,120 SO I THINK IT WOULD BE REALLY 1688 01:04:05,120 --> 01:04:07,440 HELPFUL IF NIH AND NCATS AND 1689 01:04:07,440 --> 01:04:09,120 OTHERS COULD TAKE A LEADERSHIP 1690 01:04:09,120 --> 01:04:14,360 ROLE IN EDUCATING WHAT IS THE 1691 01:04:14,360 --> 01:04:15,280 SITUATION WE HAVE NOW, WHAT ARE 1692 01:04:15,280 --> 01:04:18,640 YOU HEARING FROM CLINICIANS, AND 1693 01:04:18,640 --> 01:04:19,360 WHAT IS THE NEED. 1694 01:04:19,360 --> 01:04:20,640 WE KNOW IT WOULD BE IDEAL TO 1695 01:04:20,640 --> 01:04:24,160 COVER THE BEST POSSIBLE TEST, 1696 01:04:24,160 --> 01:04:26,120 AND IF MONEY WERE NO OBJECT ET 1697 01:04:26,120 --> 01:04:27,400 CETERA, ET CETERA BUT THE 1698 01:04:27,400 --> 01:04:29,840 REALITY IS FOR THE PATIENTS WE 1699 01:04:29,840 --> 01:04:31,320 JUST WANT THE FASTEST POSSIBLE 1700 01:04:31,320 --> 01:04:34,760 DIAGNOSIS IN OUR INDIVIDUAL 1701 01:04:34,760 --> 01:04:36,200 GENETICIST KNOW THE BEST WAY TO 1702 01:04:36,200 --> 01:04:37,160 GET THERE. 1703 01:04:37,160 --> 01:04:38,960 THEY SHOULDN'T HAVE THEIR HANDS 1704 01:04:38,960 --> 01:04:43,040 TIED WHEN IT COMES TO OBTAINING 1705 01:04:43,040 --> 01:04:45,960 DIAGNOSES AND THE COST OF 1706 01:04:45,960 --> 01:04:48,000 SEQUENCING WHILE IN THEORY HAS 1707 01:04:48,000 --> 01:04:49,360 COME WAY DOWN THE COST TO 1708 01:04:49,360 --> 01:04:50,560 PATIENT AND SYSTEM IS MUCH 1709 01:04:50,560 --> 01:04:52,480 GREATER. 1710 01:04:52,480 --> 01:04:53,920 SO WE NEED THE MAKE SURE THAT 1711 01:04:53,920 --> 01:04:57,240 ACCESS IS THERE AND CLINICS HAVE 1712 01:04:57,240 --> 01:04:58,280 DISCRETION FOR TESTS THEY DEEM 1713 01:04:58,280 --> 01:05:00,040 FIT AND WE ARE NOT LENLTS LATING 1714 01:05:00,040 --> 01:05:04,240 ANY PARTICULAR TECHNOLOGIES -- 1715 01:05:04,240 --> 01:05:04,760 LEGISLATING. 1716 01:05:04,760 --> 01:05:06,560 SO THIS IS AN ONGOING ISSUE. 1717 01:05:06,560 --> 01:05:10,160 AND THEN JUST REAL QUICK, WHAT 1718 01:05:10,160 --> 01:05:11,600 REBECCA SAID ABOUT CLINICAL 1719 01:05:11,600 --> 01:05:14,640 SUPPORT MY DAUGHTER IS FIVE NOW, 1720 01:05:14,640 --> 01:05:16,600 I HAVE NO IDEA WHAT WE WILL DO 1721 01:05:16,600 --> 01:05:18,360 WHEN SHE BECOMES AN ADULT, 1722 01:05:18,360 --> 01:05:19,640 TERRIFIES ME TO THINK ABOUT BUT 1723 01:05:19,640 --> 01:05:23,160 THAT COORDINATION OF CARE FOR A 1724 01:05:23,160 --> 01:05:25,040 FIVE-YEAR-OLD IS CHALLENGING AND 1725 01:05:25,040 --> 01:05:27,080 I SEE MARSHALL NODDING HIS HEAD 1726 01:05:27,080 --> 01:05:28,680 BUT I'M SURE CHILDREN'S NATIONAL 1727 01:05:28,680 --> 01:05:29,920 IS COMPARING FOR ME BUT WE 1728 01:05:29,920 --> 01:05:32,760 ACTUALLY FINALLY GOT INTO THE 1729 01:05:32,760 --> 01:05:34,760 COMPLEX CARE PROGRAM THERE FOR 1730 01:05:34,760 --> 01:05:36,160 CHARLOTTE, WHICH IS HUGE BECAUSE 1731 01:05:36,160 --> 01:05:38,960 WE ARE SEEING -- DIAGNOSED BY 1732 01:05:38,960 --> 01:05:41,080 MARSHALL'S TEAM IN MET BOLLICS 1733 01:05:41,080 --> 01:05:43,800 GENETICS WE ARE NOW BEING SEEN 1734 01:05:43,800 --> 01:05:46,720 BY ANDREA GROATMAN IN 1735 01:05:46,720 --> 01:05:48,280 NEUROGENETICS AND WE HAVE 1736 01:05:48,280 --> 01:05:49,480 CARDIOLOGY DEVELOPMENT, PHYSICAL 1737 01:05:49,480 --> 01:05:52,120 MEDICINE AND HA IS JUST 1738 01:05:52,120 --> 01:05:54,120 CHILDREN, THERAPIST AT HBCU SO 1739 01:05:54,120 --> 01:05:56,240 SON SO THAT COORDINATION OF CARE 1740 01:05:56,240 --> 01:05:58,120 EVEN FOR KID CHARLOTTE IS NOT 1741 01:05:58,120 --> 01:06:00,440 EVEN MEDICALLY FRAGILE, HE BRAIN 1742 01:06:00,440 --> 01:06:02,120 JUST DOESN'T TALK TO HER BODY 1743 01:06:02,120 --> 01:06:04,200 CORRECTLY SO SHE IS GOT A LEG UP 1744 01:06:04,200 --> 01:06:07,720 ON A LOT OF OTHER KIDS WHO HAVE 1745 01:06:07,720 --> 01:06:09,640 FRAGILITY IN ADDITION AND IT IS 1746 01:06:09,640 --> 01:06:10,560 UNCREDIBLY CHALLENGING, 1747 01:06:10,560 --> 01:06:11,840 BASICALLY A FULL TIME JOB SO I 1748 01:06:11,840 --> 01:06:14,600 THINK ACROSS THE BOARD WE GOT TO 1749 01:06:14,600 --> 01:06:15,520 FIGURE OUT THAT COORDINATION OF 1750 01:06:15,520 --> 01:06:16,360 CARE. 1751 01:06:16,360 --> 01:06:17,680 AND IT IS NOT JUST A MEDICAL 1752 01:06:17,680 --> 01:06:18,560 SYSTEM BUT IT IS THE EDUCATION 1753 01:06:18,560 --> 01:06:20,400 SYSTEM. 1754 01:06:20,400 --> 01:06:21,480 BECAUSE KIDS ARE RECEIVING ALL 1755 01:06:21,480 --> 01:06:26,360 THESE THERAPIES, IN THE ACADEMIC 1756 01:06:26,360 --> 01:06:27,040 SETTING AS WELL. 1757 01:06:27,040 --> 01:06:30,440 THEIR WHOLE LIFE SO MY COUPLE OF 1758 01:06:30,440 --> 01:06:33,640 COMMENTS. 1759 01:06:33,640 --> 01:06:34,840 DIAGNOSTIC IS THE BIGGEST ONE, 1760 01:06:34,840 --> 01:06:37,360 IF WE CAN'T DIAGNOSE WE CAN'T 1761 01:06:37,360 --> 01:06:38,440 GET CLINICAL TRIALS, THERAPIES 1762 01:06:38,440 --> 01:06:42,160 SO THAT S TO ME IS NUMBER ONE. 1763 01:06:42,160 --> 01:06:44,360 >> I WILL INTERRUPT AND THROW IN 1764 01:06:44,360 --> 01:06:48,720 AN AMEN. 1765 01:06:48,720 --> 01:06:50,680 >> WHILE IN COMPLETE AGREEMENT 1766 01:06:50,680 --> 01:06:51,280 WITH EVERYTHING YOU SAID AND 1767 01:06:51,280 --> 01:06:53,480 THANK YOU TO YOU FOR YOUR 1768 01:06:53,480 --> 01:06:57,360 ADVOCACY ON THIS ISSUE. 1769 01:06:57,360 --> 01:06:59,600 >> I WILL GIVE LAST WORD TO 1770 01:06:59,600 --> 01:07:01,520 KELLY AND MAKE SURE WE FOLLOW-UP 1771 01:07:01,520 --> 01:07:05,120 WITH PAUL'S IN THE CHAT TOO. 1772 01:07:05,120 --> 01:07:05,840 KELLY GO AHEAD. 1773 01:07:05,840 --> 01:07:10,040 >> THIS IS AMPLIFYING WHAT 1774 01:07:10,040 --> 01:07:11,480 KRISTINA SAID AND POINTING OUT, 1775 01:07:11,480 --> 01:07:13,560 I LOVED HEARING THAT YOU 1776 01:07:13,560 --> 01:07:15,760 DESCRIBED A BIG OPPORTUNITY 1777 01:07:15,760 --> 01:07:24,280 FOCUS ON DIAGNOSTICS I REPRESENT 1778 01:07:24,280 --> 01:07:26,480 THE LUPUS CLINICAL INVESTIGATORS 1779 01:07:26,480 --> 01:07:28,760 NETWORK WE STOOD UP THIS 57 1780 01:07:28,760 --> 01:07:29,880 MEDICAL CENTER CLINICAL TRIAL 1781 01:07:29,880 --> 01:07:34,000 NETWORK FIVE YEARS AGO AS A 1782 01:07:34,000 --> 01:07:36,640 RESOURCE FOR PHARMA AND OTHER 1783 01:07:36,640 --> 01:07:38,680 ORGANIZATIONS TO ACTIVATE SO 1784 01:07:38,680 --> 01:07:40,640 THEY CAN GET REALLY WELL 1785 01:07:40,640 --> 01:07:42,840 CONTROLLED PATIENTS AND WHAT IS 1786 01:07:42,840 --> 01:07:46,240 REALLY INTERESTING AS A PATIENT 1787 01:07:46,240 --> 01:07:47,480 MYSELF, WE HAVE A POPULATION 1788 01:07:47,480 --> 01:07:49,240 WHERE TIME TO DIAGNOSIS IS TEN 1789 01:07:49,240 --> 01:07:50,840 PLUS YEARS UNLESS YOU HAVE THE 1790 01:07:50,840 --> 01:07:54,640 MOST SEVERE CASE, IT IS A VERY 1791 01:07:54,640 --> 01:07:57,240 HETEROGENOUS DISEASE AND PHARMA 1792 01:07:57,240 --> 01:07:59,560 WITH DRUG AND OTHER TYPE OF 1793 01:07:59,560 --> 01:08:00,880 THERAPEUTIC INTERVENTION, HAVE 1794 01:08:00,880 --> 01:08:04,560 RUSHED TO ACTIVATE OUR CLINICAL 1795 01:08:04,560 --> 01:08:06,160 TRIAL NETWORK WE CANNOT 1796 01:08:06,160 --> 01:08:09,800 INCENTIVIZE FOR THE LIFE OF US, 1797 01:08:09,800 --> 01:08:11,160 DIAGNOSTIC COMPANIES TO ACTIVATE 1798 01:08:11,160 --> 01:08:11,640 OUR NETWORK. 1799 01:08:11,640 --> 01:08:13,880 IT IS A RESOURCE THAT COSTS 1800 01:08:13,880 --> 01:08:16,360 ALMOST NOTHING SO IT IS AN 1801 01:08:16,360 --> 01:08:20,680 ALMOST FREE ACTIVITY AND IT 1802 01:08:20,680 --> 01:08:24,000 COMES WITH 200 PIs OPERATING 1803 01:08:24,000 --> 01:08:25,760 UNDER A COMMON IRB SO IT'S 1804 01:08:25,760 --> 01:08:30,320 BAFFLED ME AS A PATIENT, THAT WE 1805 01:08:30,320 --> 01:08:32,000 CAN'T GIVE AWAY THIS ALMOST FREE 1806 01:08:32,000 --> 01:08:32,880 RESOURCE, IT SAYS SOMETHING 1807 01:08:32,880 --> 01:08:35,200 ABOUT THE BUSINESS MODEL THAT I 1808 01:08:35,200 --> 01:08:36,640 GUESS MAYBE BEGS A POLICY 1809 01:08:36,640 --> 01:08:40,200 QUESTION, MAYBE THERE NEEDS TO 1810 01:08:40,200 --> 01:08:43,360 BE SOME CONVERSATION AS YOU GUYS 1811 01:08:43,360 --> 01:08:45,360 SEE THIS OPPORTUNITY AND WANT TO 1812 01:08:45,360 --> 01:08:48,880 HARNESS THIS OPPORTUNITY IN 1813 01:08:48,880 --> 01:08:50,400 DIAGNOSTICS, AS KRISTINA SAYS 1814 01:08:50,400 --> 01:08:51,520 THERE NEEDS TO BE EDUCATION ON 1815 01:08:51,520 --> 01:08:53,040 LOTS OF LEVELS BUT THERE 1816 01:08:53,040 --> 01:08:55,120 PROBABLY NEEDS TO BE SOME KIND 1817 01:08:55,120 --> 01:08:58,840 OF INDUSTRY INCENTIVES TO SEE 1818 01:08:58,840 --> 01:09:02,080 SOME COMMERCIAL VALUE BECAUSE 1819 01:09:02,080 --> 01:09:07,000 AGAIN WE ARE OFFERING THIS UP, 1820 01:09:07,000 --> 01:09:08,760 PHARMA IS KNOCKING ON THE DOOR 1821 01:09:08,760 --> 01:09:10,000 FOR THE CLINICAL TRIAL NETWORK, 1822 01:09:10,000 --> 01:09:11,440 WE CAN'T GET DIAGNOSTIC 1823 01:09:11,440 --> 01:09:14,240 COMPANIES TO USE IT. 1824 01:09:14,240 --> 01:09:15,320 FOR WHAT -- 1825 01:09:15,320 --> 01:09:17,600 >> I THINK THAT'S VERY FAIR, 1826 01:09:17,600 --> 01:09:19,160 THAT DIAGNOSIS ISN'T JUST A RARE 1827 01:09:19,160 --> 01:09:20,320 DISEASE ISSUE. 1828 01:09:20,320 --> 01:09:22,600 THERE ARE PLENTY OF COMMON 1829 01:09:22,600 --> 01:09:24,640 DISEASES THAT'S SPECIALLY IF THE 1830 01:09:24,640 --> 01:09:26,600 ATYPICAL PRESENTATIONS ARE 1831 01:09:26,600 --> 01:09:27,080 DIAGNOSED TOO. 1832 01:09:27,080 --> 01:09:28,880 IF YOU ARE UNDIAGNOSED WE DON'T 1833 01:09:28,880 --> 01:09:30,480 KNOW WHAT YOU HAVE SO COULD BE 1834 01:09:30,480 --> 01:09:31,880 COMMON OR RARE DISEASE. 1835 01:09:31,880 --> 01:09:34,160 SO I THINK THINGS LIKE THIS 1836 01:09:34,160 --> 01:09:35,640 COULD HELP REALLY LITERALLY 1837 01:09:35,640 --> 01:09:37,880 EVERYONE. 1838 01:09:37,880 --> 01:09:39,840 I THINK THAT IS RIGHT. 1839 01:09:39,840 --> 01:09:41,600 >> WE BELIEVE THAT A LOT OF OUR 1840 01:09:41,600 --> 01:09:43,360 PATIENTS PROBABLY DO HAVE 1841 01:09:43,360 --> 01:09:44,400 UNDIAGNOSED RARE DISEASE THAT 1842 01:09:44,400 --> 01:09:46,760 JUST HAVEN'T BEEN IDENTIFIED YET 1843 01:09:46,760 --> 01:09:48,760 BECAUSE IT IS TOO HETEROGENOUS 1844 01:09:48,760 --> 01:09:50,200 TO IN THE FUTURE HAVE IT BE 1845 01:09:50,200 --> 01:09:52,240 UNDER THIS ONE COMMON UMBRELLA. 1846 01:09:52,240 --> 01:09:55,240 SO THAT IS SEPARATE TOPIC. 1847 01:09:55,240 --> 01:09:58,280 >> THANK YOU. 1848 01:09:58,280 --> 01:10:00,480 SO MAYBE WE WILL DO ONE MORE 1849 01:10:00,480 --> 01:10:02,000 MATTHIAS THEN WE IMMEDIATE TO 1850 01:10:02,000 --> 01:10:04,480 MOVE ON TOO. 1851 01:10:04,480 --> 01:10:05,080 MATTHIAS. 1852 01:10:05,080 --> 01:10:07,040 >> REAL QUICK. 1853 01:10:07,040 --> 01:10:08,240 JUST RECOMMENDATION FOR 1854 01:10:08,240 --> 01:10:11,680 CONTINUING NOT TO LOSE FOCUS, 1855 01:10:11,680 --> 01:10:14,880 RARE DISEASE SYSTEM NO -- AND 1856 01:10:14,880 --> 01:10:18,200 NCATS IS ACTIVE IN IRDRDC AND I 1857 01:10:18,200 --> 01:10:22,040 WANT TO ENCOURAGE YOU DESPITE 1858 01:10:22,040 --> 01:10:25,080 SOME COST FROM THE HILL THAT WE 1859 01:10:25,080 --> 01:10:28,160 CANNOT HAVE INHIBIT -- WHICH IS 1860 01:10:28,160 --> 01:10:30,000 THE (INAUDIBLE) WE ENCOURAGE I 1861 01:10:30,000 --> 01:10:31,960 KNOW PJ HAS LEADERSHIP FUNCTION 1862 01:10:31,960 --> 01:10:34,240 WITH IRDRC AND AT LEAST CONTINUE 1863 01:10:34,240 --> 01:10:37,560 THAT WORK AND SEND CLEAR 1864 01:10:37,560 --> 01:10:39,240 MESSAGES TO OUR SCIENTISTS IN 1865 01:10:39,240 --> 01:10:42,440 THE TRENCHES, THAT WE ARE 1866 01:10:42,440 --> 01:10:43,440 ENCOURAGE OBVIOUSLY IN THE 1867 01:10:43,440 --> 01:10:46,080 FRAMEWORK WHICH IS PROVIDED BY 1868 01:10:46,080 --> 01:10:49,520 LEGAL GUIDANCE TO US THAT WE ARE 1869 01:10:49,520 --> 01:10:51,160 ENCOURAGED TO CONTINUE TO 1870 01:10:51,160 --> 01:10:52,840 ADVANCE RARE DISEASE IN GLOBAL 1871 01:10:52,840 --> 01:10:53,960 MANNER, THIS IS A SENSITIVE 1872 01:10:53,960 --> 01:10:55,800 ISSUE, THAT IS WHY I PUT IT UP 1873 01:10:55,800 --> 01:11:00,760 TO THIS FORUM, VERY MUCH 1874 01:11:00,760 --> 01:11:02,360 APPRECIATE HIGH LEVEL CONTINUED 1875 01:11:02,360 --> 01:11:03,760 EFFORTS FROM NCATS IN THIS 1876 01:11:03,760 --> 01:11:04,320 SPACE. 1877 01:11:04,320 --> 01:11:05,800 >> THANKS, MATTHIAS. 1878 01:11:05,800 --> 01:11:07,600 I KNOW MARSHALL I DON'T WANT THE 1879 01:11:07,600 --> 01:11:10,040 SPEAK FOR YOU BUT I KNOW YOU 1880 01:11:10,040 --> 01:11:11,200 ECHO THAT SENT M AS WELL. 1881 01:11:11,200 --> 01:11:13,960 GOOD POINT. 1882 01:11:13,960 --> 01:11:15,480 AND WE WILL CONTINUE TO DO THAT. 1883 01:11:15,480 --> 01:11:17,800 >> SO MANY ACTUAL EFFORTS AND WE 1884 01:11:17,800 --> 01:11:20,680 CANNOT AFFORD TO WASTE A SINGLE 1885 01:11:20,680 --> 01:11:22,240 PATIENT TO HELP ADD TO THE 1886 01:11:22,240 --> 01:11:23,720 KNOWLEDGE WE HAVE, MOVING THESE 1887 01:11:23,720 --> 01:11:26,880 FORWARD. 1888 01:11:26,880 --> 01:11:28,680 IT IS REALLY A GLOBAL EXERCISE. 1889 01:11:28,680 --> 01:11:29,480 >> THANK YOU. 1890 01:11:29,480 --> 01:11:33,480 BEFORE WE MOVE ON TO THE CONCEPT 1891 01:11:33,480 --> 01:11:34,960 CLEARANCE PAUL HARRIS ASKED A 1892 01:11:34,960 --> 01:11:37,920 QUESTION ABOUT GARD 2.0 AND WHEN 1893 01:11:37,920 --> 01:11:39,840 THAT IS COMING OUT. 1894 01:11:39,840 --> 01:11:41,240 ANNE AN OVERVIEW OF THAT TIME 1895 01:11:41,240 --> 01:11:44,600 FRAME. 1896 01:11:44,600 --> 01:11:47,440 >> THE TEST SITE IS UP NOW YOU 1897 01:11:47,440 --> 01:11:48,560 CAN ACCESS NOW. 1898 01:11:48,560 --> 01:11:51,760 WHEN WE WILL BE ABLE TO FULLY 1899 01:11:51,760 --> 01:11:53,000 LAUNCH 2.0 DEPENDS A LITTLE BIT 1900 01:11:53,000 --> 01:11:55,840 ON THE EXTENT OF WHAT FEEDBACK 1901 01:11:55,840 --> 01:11:57,600 WE GET AND WHAT NEEDS TO BE 1902 01:11:57,600 --> 01:12:00,080 FIXED. 1903 01:12:00,080 --> 01:12:02,160 WE HOPE IT WILL BE IN THE NEAR 1904 01:12:02,160 --> 01:12:04,960 TERM RATHER THAN THE LONGER TERM 1905 01:12:04,960 --> 01:12:07,360 BUT WE ARE CONTINUING TO LOOK. 1906 01:12:07,360 --> 01:12:08,280 >> THANK YOU. 1907 01:12:08,280 --> 01:12:10,440 YOU WILL CONVERT SPANISH AS 1908 01:12:10,440 --> 01:12:10,960 WELL? 1909 01:12:10,960 --> 01:12:14,200 I CHECKED OUT THE VERSION 1910 01:12:14,200 --> 01:12:15,640 AVAILABLE ON THE WEBSITE AND IT 1911 01:12:15,640 --> 01:12:17,040 LOOKS GOOD BUT IT HAS HOLES AND 1912 01:12:17,040 --> 01:12:18,680 I WAS WONDERING ABOUT THE USER 1913 01:12:18,680 --> 01:12:20,560 EXPERIENCE TESTING AS WELL AS 1914 01:12:20,560 --> 01:12:22,560 THE MULTI-LINGUAL. 1915 01:12:22,560 --> 01:12:25,440 >> IT HAS SOME HOLES FOR THE 1916 01:12:25,440 --> 01:12:26,960 INITIAL TEST LAUNCH, IT IS 1917 01:12:26,960 --> 01:12:28,760 ENGLISH ONLY BUT THE PLANS FOR 1918 01:12:28,760 --> 01:12:30,520 THE SPANISH ARE THERE. 1919 01:12:30,520 --> 01:12:31,200 >> OKAY. 1920 01:12:31,200 --> 01:12:34,800 THANKS. 1921 01:12:34,800 --> 01:12:37,040 >> WITH THAT WE ARE A LITTLE 1922 01:12:37,040 --> 01:12:38,560 BEHIND TIME BUT OKAY THIS IS 1923 01:12:38,560 --> 01:12:40,200 GOING TO WORK FLUENTLY INTO 1924 01:12:40,200 --> 01:12:41,560 CONCEPT CLEARANCE BECAUSE IT 1925 01:12:41,560 --> 01:12:44,040 DOES HAVE TO DO WITH RDCRN AND 1926 01:12:44,040 --> 01:12:45,360 PROJECTS WITHIN THAT. 1927 01:12:45,360 --> 01:12:47,680 ANNE ARE YOU GIVING A BRIEF 1928 01:12:47,680 --> 01:12:48,880 OVERVIEW BEFORE TIINA TAKES 1929 01:12:48,880 --> 01:12:49,360 OVER? 1930 01:12:49,360 --> 01:12:52,800 GO AHEAD. 1931 01:12:52,800 --> 01:12:59,400 >> ANOTHER TAG TEAM. 1932 01:12:59,400 --> 01:13:01,360 SO I JUST SHOWED Y'ALL THIS SO I 1933 01:13:01,360 --> 01:13:02,920 WON'T SPEND TIME ON IT BUT IT IS 1934 01:13:02,920 --> 01:13:04,520 AGAIN JUST TO PUT THIS IN 1935 01:13:04,520 --> 01:13:09,840 CONTEXT THE RDCRN IS OUR LARGEST 1936 01:13:09,840 --> 01:13:11,440 RESEARCH ARMS BUT FITS THIS 1937 01:13:11,440 --> 01:13:12,560 LARGER PICTURE OF TRYING TO 1938 01:13:12,560 --> 01:13:16,880 IMPROVE THE RESEARCH ENVIRONMENT 1939 01:13:16,880 --> 01:13:17,920 FOR PATIENTS. 1940 01:13:17,920 --> 01:13:22,120 NEXT PLEASE. 1941 01:13:22,120 --> 01:13:23,680 SO JUST BEFORE WE SHOWED THIS 1942 01:13:23,680 --> 01:13:24,960 BEFORE AS WELL, THIS IS KIND OF 1943 01:13:24,960 --> 01:13:30,040 A GRAPHIC OF THE CURRENT RDCRN 4 1944 01:13:30,040 --> 01:13:32,240 AND BIT OF A BUSY SLIDE BUT HERE 1945 01:13:32,240 --> 01:13:34,360 ARE THE 20 CONSORTIA, YOU CAN 1946 01:13:34,360 --> 01:13:35,960 APPRECIATE THEY ARE VERY DIVERSE 1947 01:13:35,960 --> 01:13:37,880 YET THEY MANAGE TO WORK TOGETHER 1948 01:13:37,880 --> 01:13:40,760 EXTREMELY WELL TO SHARE SNOJ AND 1949 01:13:40,760 --> 01:13:43,320 INFORMATION AND SHARE 1950 01:13:43,320 --> 01:13:45,360 EXPERIENCES BUT ALSO A 1951 01:13:45,360 --> 01:13:47,680 COLLABORATION WITH TEN NIH 1952 01:13:47,680 --> 01:13:50,760 INSTITUTES AND OFFICES RESEARCH 1953 01:13:50,760 --> 01:13:52,320 CLINICIANS ACADEMIC MEDICAL 1954 01:13:52,320 --> 01:13:54,280 CENTERS AS WELL AS LEADERSHIP 1955 01:13:54,280 --> 01:13:56,040 THAT IS SHOWN BY PATIENTS AND 1956 01:13:56,040 --> 01:13:58,560 PATIENT ADVOCACY GROUPS. 1957 01:13:58,560 --> 01:14:00,480 INSTITUTES ARE ALL LISTED THERE 1958 01:14:00,480 --> 01:14:03,360 AT BOTTOM. 1959 01:14:03,360 --> 01:14:04,520 NEXT PLEASE. 1960 01:14:04,520 --> 01:14:07,920 SO WE WILL BE TALKING ABOUT TWO 1961 01:14:07,920 --> 01:14:09,400 PROGRAM COMPONENTS WE DISCUSS AS 1962 01:14:09,400 --> 01:14:13,120 PART OF THE RDCRN, SHOWN UP IN 1963 01:14:13,120 --> 01:14:14,640 PREVIOUS ITERATIONS, TWO 1964 01:14:14,640 --> 01:14:15,960 SEPARATE FUNDING ANNOUNCEMENTS 1965 01:14:15,960 --> 01:14:17,880 WHICH IS WHY WE ARE DOING THEM 1966 01:14:17,880 --> 01:14:18,360 SEPARATELY. 1967 01:14:18,360 --> 01:14:20,760 SO THE FIRST ONE IS FOR THE RARE 1968 01:14:20,760 --> 01:14:22,480 DISEASE CLINICAL RESEARCH 1969 01:14:22,480 --> 01:14:25,360 CONSORTIA OR THE RDCRC AND OTHER 1970 01:14:25,360 --> 01:14:26,760 IS FOR DATA MANAGEMENT 1971 01:14:26,760 --> 01:14:28,160 COORDINATING CENTER WHICH IS THE 1972 01:14:28,160 --> 01:14:30,840 NAME IMPLIES KIND OF HOLDS THE 1973 01:14:30,840 --> 01:14:32,520 NETWORK TOGETHER AND COORDINATES 1974 01:14:32,520 --> 01:14:37,360 EFFORTS AS WELL AS DEA. 1975 01:14:37,360 --> 01:14:39,040 SO THE -- TIINA WILL BE 1976 01:14:39,040 --> 01:14:40,880 PRESENTING THE CONCEPT, AND 1977 01:14:40,880 --> 01:14:44,720 SHE'S GOING TO BE GOING -- DOING 1978 01:14:44,720 --> 01:14:46,240 TOGETHER BECAUSE GOALS AND 1979 01:14:46,240 --> 01:14:47,800 OVERVIEW APPLY TO BOTH CONCEPTS 1980 01:14:47,800 --> 01:14:49,000 SO JUST STORE THESE IN YOUR 1981 01:14:49,000 --> 01:14:50,480 MEMORY AS YOU ARE LISTENING. 1982 01:14:50,480 --> 01:14:52,680 AND THIS WILL THEN BE FOLLOWED 1983 01:14:52,680 --> 01:14:54,160 BY INDIVIDUAL CONSIDERATION FOR 1984 01:14:54,160 --> 01:14:57,080 EACH OF THE CONCEPTS BECAUSE 1985 01:14:57,080 --> 01:14:59,600 THERE ARE SOME DIFFERENCES, 1986 01:14:59,600 --> 01:15:01,440 BETWEEN THEM. 1987 01:15:01,440 --> 01:15:03,440 SO THERE WILL BE OPPORTUNITY FOR 1988 01:15:03,440 --> 01:15:04,560 QUESTIONS AND COMMENTS AFTER 1989 01:15:04,560 --> 01:15:07,080 EACH OF THE SEPARATE CONCEPTS. 1990 01:15:07,080 --> 01:15:08,400 AND THEY WILL BE VOTED ON 1991 01:15:08,400 --> 01:15:08,760 SEPARATELY. 1992 01:15:08,760 --> 01:15:11,000 AS WELL. 1993 01:15:11,000 --> 01:15:13,240 IF THEY ARE APPROVED BY COUNCIL 1994 01:15:13,240 --> 01:15:14,960 THEY WOULD HAVE SEPARATE FUNDING 1995 01:15:14,960 --> 01:15:16,280 ANNOUNCEMENTS SO I WANT TO 1996 01:15:16,280 --> 01:15:18,360 CLARIFY THAT BEFORE TIINA JUMPS 1997 01:15:18,360 --> 01:15:18,880 IN. 1998 01:15:18,880 --> 01:15:21,400 NEXT PLEASE. 1999 01:15:21,400 --> 01:15:27,120 HAND THIS OVER TO TIINA. 2000 01:15:27,120 --> 01:15:29,960 >> THANK YOU VERY MUCH, REALLY 2001 01:15:29,960 --> 01:15:31,520 APPRECIATE THE SETTING THE STAGE 2002 01:15:31,520 --> 01:15:32,880 FOR WHAT I'M GOING TO BE TALKING 2003 01:15:32,880 --> 01:15:34,520 ABOUT, CAN EVERYONE HEAR ME ALL 2004 01:15:34,520 --> 01:15:37,080 RIGHT? 2005 01:15:37,080 --> 01:15:39,080 SO WHAT I AM GOING TO DO IS LIKE 2006 01:15:39,080 --> 01:15:41,960 ANNE SAID I WILL TALK ABOUT THE 2007 01:15:41,960 --> 01:15:45,640 RDCR AND CLINICAL RESEARCH 2008 01:15:45,640 --> 01:15:47,880 CONSORTIA FIRST AND WE WILL TAKE 2009 01:15:47,880 --> 01:15:49,560 SOME QUESTIONS, THEN TALK ABOUT 2010 01:15:49,560 --> 01:15:51,080 DATA MANAGEMENT AND COORDINATING 2011 01:15:51,080 --> 01:15:52,320 CENTER AND TAKE SOME QUESTIONS. 2012 01:15:52,320 --> 01:15:55,960 NEXT SLIDE PLEASE. 2013 01:15:55,960 --> 01:15:57,640 SOME WILL BE REDUNDANT SINCE WE 2014 01:15:57,640 --> 01:16:00,720 HAD OUR WONDERFUL ORDR 2015 01:16:00,720 --> 01:16:04,680 PRESENTATION BEFORE THIS, SO THE 2016 01:16:04,680 --> 01:16:06,000 RDCRN WAS ESTABLISHED BY RARE 2017 01:16:06,000 --> 01:16:07,360 DISEASE ABLG OF TWOA AND THE 2018 01:16:07,360 --> 01:16:08,920 PURPOSE WAS TO -- 2002 AND THE 2019 01:16:08,920 --> 01:16:11,240 PURPOSE WAS FOR A COLLABORATIVE 2020 01:16:11,240 --> 01:16:13,760 MULTI-SITE PATIENT CENTRIC 2021 01:16:13,760 --> 01:16:14,560 TRANSLATIONAL CLINICAL TRIAL FOR 2022 01:16:14,560 --> 01:16:15,920 RESEARCH IN RARE DISEASES. 2023 01:16:15,920 --> 01:16:18,560 AND IF YOU THINK BACK TO 2002 2024 01:16:18,560 --> 01:16:20,160 THERE WAS REALLY NOTHING LIKE 2025 01:16:20,160 --> 01:16:24,000 THIS GOING ON. 2026 01:16:24,000 --> 01:16:25,920 THIS -- THE STUDY OF THREE 2027 01:16:25,920 --> 01:16:27,480 DISEASES OR MORE AT A TIME GOES 2028 01:16:27,480 --> 01:16:30,120 ALL THE WAY BACK TO 2002. 2029 01:16:30,120 --> 01:16:33,360 SO THIS WILL FOCUS OF ORDR IN 2030 01:16:33,360 --> 01:16:36,840 THE RARE DISEASE CLINICAL 2031 01:16:36,840 --> 01:16:37,920 RESEARCH NETWORK. 2032 01:16:37,920 --> 01:16:40,160 THE GAPS WE TRYING TO FILL IN 2033 01:16:40,160 --> 01:16:41,440 THIS PROGRAM IS LACK OF 2034 01:16:41,440 --> 01:16:42,480 FOUNDATIONAL TRANSLATIONAL 2035 01:16:42,480 --> 01:16:44,360 RESEARCH IN RARE DISEASES, WE 2036 01:16:44,360 --> 01:16:45,600 NEED MORE INFORMATION FULLY 2037 01:16:45,600 --> 01:16:48,000 DESCRIBES DEES AND FACILITATES 2038 01:16:48,000 --> 01:16:49,560 MOVEMENT OF CANDIDATES TOWARDS 2039 01:16:49,560 --> 01:16:50,640 TREATMENT. 2040 01:16:50,640 --> 01:16:52,760 THERE IS A HIGH COST OF ONE 2041 01:16:52,760 --> 01:16:54,760 DISEASE AT A TIME APPROACH, AND 2042 01:16:54,760 --> 01:16:57,400 THIS IS IN BOTH TIME AND MONEY 2043 01:16:57,400 --> 01:16:59,920 THERE IS A LACK OF STANDARDIZED 2044 01:16:59,920 --> 01:17:01,640 HIGH QUALITY DATA IN BROAD DATA 2045 01:17:01,640 --> 01:17:04,200 SHARING FOR RARE DISEASES. 2046 01:17:04,200 --> 01:17:07,160 SO NCATS PROPOSES CONCEPT TO 2047 01:17:07,160 --> 01:17:08,520 ADVANCE DIAGNOSIS MANAGEMENT AND 2048 01:17:08,520 --> 01:17:10,520 TREATMENT OF RARE DISEASES BY 2049 01:17:10,520 --> 01:17:13,720 FOCUSING ON UNMET CLINICAL TRIAL 2050 01:17:13,720 --> 01:17:15,280 READINESS NEEDS THAT WILL MOVE 2051 01:17:15,280 --> 01:17:16,760 THE FEERLD OF RESEARCH FORWARD 2052 01:17:16,760 --> 01:17:17,920 FROM CURRENT STATE BY 2053 01:17:17,920 --> 01:17:20,400 ESTABLISHING MAINTAINING AND 2054 01:17:20,400 --> 01:17:22,120 SUPPORTING RARE DISEASE CENTERS 2055 01:17:22,120 --> 01:17:22,920 OF EXCELLENCE, NEXT SLIDE 2056 01:17:22,920 --> 01:17:25,440 PLEASE. 2057 01:17:25,440 --> 01:17:27,320 SO THE OBJECTIVES WE HAVE. 2058 01:17:27,320 --> 01:17:29,080 WHAT WE WANT -- TRYING TO 2059 01:17:29,080 --> 01:17:32,480 ACCOMPLISH IS REALLY LESSEN RISK 2060 01:17:32,480 --> 01:17:34,360 OF FAILURE IN CLINICAL TRIALS BY 2061 01:17:34,360 --> 01:17:36,240 BEING WELL PREPARED OR WHAT WE 2062 01:17:36,240 --> 01:17:38,760 REFER TO OFTEN AS CLINICAL TRIAL 2063 01:17:38,760 --> 01:17:40,920 READY PRIOR TO STARTING A 2064 01:17:40,920 --> 01:17:41,360 CLINICAL TRIAL. 2065 01:17:41,360 --> 01:17:43,360 WE WANT TO DEVELOP AN EFFECTIVE 2066 01:17:43,360 --> 01:17:46,040 AND EFFICIENT RESEARCH 2067 01:17:46,040 --> 01:17:47,560 ENVIRONMENT FOR RARE DISEASE 2068 01:17:47,560 --> 01:17:48,920 RESEARCH AND WE ARE THINKING OF 2069 01:17:48,920 --> 01:17:52,160 THIS AS A RESEARCH COMMUNITY FOR 2070 01:17:52,160 --> 01:17:52,760 RARE DISEASE. 2071 01:17:52,760 --> 01:17:56,240 WE WANT TO WORK FASTER HOW? 2072 01:17:56,240 --> 01:17:58,640 BY ESTABLISHING CONSORTIA OF 2073 01:17:58,640 --> 01:17:59,840 INVESTIGATORS AND PATIENT GROUPS 2074 01:17:59,840 --> 01:18:01,560 AND HAVING LONGITUDINAL STUDIES 2075 01:18:01,560 --> 01:18:02,320 TO DEVELOP A BETTER 2076 01:18:02,320 --> 01:18:05,920 UNDERSTANDING OF THE DISEASE SO 2077 01:18:05,920 --> 01:18:07,160 WHEN IT IS TIME FOR A CLINICAL 2078 01:18:07,160 --> 01:18:08,520 TRIAL, THAT WILL ALREADY BE IN 2079 01:18:08,520 --> 01:18:09,000 PLACE. 2080 01:18:09,000 --> 01:18:11,000 WE WANT TO WORK IN A COST 2081 01:18:11,000 --> 01:18:13,000 EFFECTIVE MANNER REALLY FOCUSING 2082 01:18:13,000 --> 01:18:15,200 ON ECONOMIES OF SCALE, BY USING 2083 01:18:15,200 --> 01:18:16,960 SHARED TOOLS IN THE WORK 2084 01:18:16,960 --> 01:18:17,280 ENVIRONMENT. 2085 01:18:17,280 --> 01:18:19,000 WHILE THE DISEASES THAT ANNE 2086 01:18:19,000 --> 01:18:21,240 POINTED OUT ARE ALL DIVERSE, 2087 01:18:21,240 --> 01:18:22,880 THERE ARE COMMONALITIES ACROSS 2088 01:18:22,880 --> 01:18:24,760 ALL THE RARE DISEASES, THAT THEY 2089 01:18:24,760 --> 01:18:27,240 CAN BE WORKING ON AS A GROUP TO 2090 01:18:27,240 --> 01:18:27,760 ADDRESS. 2091 01:18:27,760 --> 01:18:30,520 AND TO TARGET. 2092 01:18:30,520 --> 01:18:32,640 ALSO HIGH QUALITY RESEARCH DATA, 2093 01:18:32,640 --> 01:18:34,880 AND EMPHASIS ON SCIENTIFIC RIGOR 2094 01:18:34,880 --> 01:18:37,880 FAIR PRINCIPLES AND DATA 2095 01:18:37,880 --> 01:18:38,400 MANAGEMENT. 2096 01:18:38,400 --> 01:18:39,920 IF YOU START WITH GOOD DATA IN 2097 01:18:39,920 --> 01:18:41,560 THE VERY BEGINNING OF A STUDY BY 2098 01:18:41,560 --> 01:18:43,080 THE TIME YOU GET TO CLINICAL 2099 01:18:43,080 --> 01:18:44,840 TRIALS YOU WILL BE BRINGING THAT 2100 01:18:44,840 --> 01:18:46,760 STRONG DATA WITH YOU. 2101 01:18:46,760 --> 01:18:48,400 WE WANT TO FACILITATE DATA 2102 01:18:48,400 --> 01:18:49,720 SHARING AND WORKING 2103 01:18:49,720 --> 01:18:51,520 COLLABORATIVELY WITH THE RDCRN 2104 01:18:51,520 --> 01:18:53,400 DATA MANAGEMENT COORDINATING 2105 01:18:53,400 --> 01:18:53,840 CENTER. 2106 01:18:53,840 --> 01:18:56,040 KEY AREAS WE WILL BE WORKING ON 2107 01:18:56,040 --> 01:18:57,960 ARE TO CONDUCT STUDIES THAT 2108 01:18:57,960 --> 01:18:59,560 PROMOTE CLINICAL TRIAL 2109 01:18:59,560 --> 01:19:02,200 READINESS, FOR EXAMPLE, NATURAL 2110 01:19:02,200 --> 01:19:04,320 HISTORY STUDIES, DEVELOPING 2111 01:19:04,320 --> 01:19:05,480 BIOMARKERS AND DEVELOPING 2112 01:19:05,480 --> 01:19:08,080 OUTCOME MEASURES, PROVIDE 2113 01:19:08,080 --> 01:19:09,240 RESEARCH ENVIRONMENT THAT 2114 01:19:09,240 --> 01:19:10,320 PROVIDES CORE OPPORTUNITIES FOR 2115 01:19:10,320 --> 01:19:11,160 STUDENTS IN EARLY STAGE 2116 01:19:11,160 --> 01:19:12,000 INVESTIGATORS. 2117 01:19:12,000 --> 01:19:14,600 WE REALLY WANT TO MOVE ON THE 2118 01:19:14,600 --> 01:19:16,440 NEXT GENERATION OF RARE DISEASE 2119 01:19:16,440 --> 01:19:18,720 RESEARCHERS, WE STARTED IN 2002 2120 01:19:18,720 --> 01:19:20,560 AND WE LIKE TO SEE THE PEOPLE 2121 01:19:20,560 --> 01:19:22,640 COMING THROUGH AND HAVING THEIR 2122 01:19:22,640 --> 01:19:24,000 OWN GRANTS. 2123 01:19:24,000 --> 01:19:26,280 ENGAGE PATIENT A ADVOCACY GROUPS 2124 01:19:26,280 --> 01:19:27,760 AS FULL PARTNERS WITHIN THE 2125 01:19:27,760 --> 01:19:29,360 NETWORK AND WE ARE LOOKING FOR 2126 01:19:29,360 --> 01:19:30,360 NOT JUST A LETTER OF SUPPORT 2127 01:19:30,360 --> 01:19:34,200 MANY THE DOCUMENT BUT REALLY 2128 01:19:34,200 --> 01:19:36,640 BRINGING THE PATIENT ADVOCACY 2129 01:19:36,640 --> 01:19:38,160 GROUPS TO TABLE WHERE DECISIONS 2130 01:19:38,160 --> 01:19:39,760 ARE BEING MADE AND DEVELOP 2131 01:19:39,760 --> 01:19:41,920 MAINTAIN A CLOUD BASED RESEARCH 2132 01:19:41,920 --> 01:19:46,120 ENVIRONMENT WITH SHARED TOOLINGS SHARED TO OLS 2133 01:19:46,120 --> 01:19:47,720 ACROSS NETWORK COLLABORATIONS. 2134 01:19:47,720 --> 01:19:48,920 THANK YOU. 2135 01:19:48,920 --> 01:19:52,240 THE RDCRN SUPPORTS 20 RARE 2136 01:19:52,240 --> 01:19:55,080 DISEASE CONSORTIA THAT STUDY 165 2137 01:19:55,080 --> 01:19:59,680 RARE DISEASES, CONDUCTS RESEARCH 2138 01:19:59,680 --> 01:20:03,920 AT 358 SITES AND 144 CO-LOCATED 2139 01:20:03,920 --> 01:20:07,600 AT CTSA SITES AND WE ARE ALSO IN 2140 01:20:07,600 --> 01:20:09,080 12 COUNTRIES. 2141 01:20:09,080 --> 01:20:12,160 166 OF THE COALITION OF PATIENT 2142 01:20:12,160 --> 01:20:12,840 ADVOCACY GROUPS PARTNERING 2143 01:20:12,840 --> 01:20:15,920 MEMBERS OF THE CONSORTIA AND IN 2144 01:20:15,920 --> 01:20:18,880 THIS ROUND AFTER RDCRN 4 RAZ WE 2145 01:20:18,880 --> 01:20:21,000 CALL IT, TEN OF EXISTING 2146 01:20:21,000 --> 01:20:24,800 CONSORTIA WILL SUNSET AFTER 15 2147 01:20:24,800 --> 01:20:26,560 OR MORE YEARS OF RESOURCES AND 2148 01:20:26,560 --> 01:20:28,560 TEN WILL BE ELIGIBLE TO 2149 01:20:28,560 --> 01:20:29,160 RECOMPETE. 2150 01:20:29,160 --> 01:20:33,560 SO THE ORDR AND NCATS IS 2151 01:20:33,560 --> 01:20:35,440 FACILITATOR AND KOORD NAY TO OF 2152 01:20:35,440 --> 01:20:36,920 RARE DISEASE RESEARCH AT THE NIH 2153 01:20:36,920 --> 01:20:40,560 AND DISORDERS TOUCH ON MULTIPLE 2154 01:20:40,560 --> 01:20:41,560 DIFFERENT INSTITUTES AND THERE 2155 01:20:41,560 --> 01:20:44,840 IS INTEREST ACROSS THEM. 2156 01:20:44,840 --> 01:20:46,320 SO WE ARE PARTNERS, CLOSE 2157 01:20:46,320 --> 01:20:48,600 PARTNERS WITH NINE OTHER NIH 2158 01:20:48,600 --> 01:20:49,000 ICs. 2159 01:20:49,000 --> 01:20:51,880 THE RDCRN IS ONE OF A KIND 2160 01:20:51,880 --> 01:20:53,440 TRANSLATIONAL RESEARCH NETWORK 2161 01:20:53,440 --> 01:20:55,000 AT NIH AND WE ESTABLISHED 2162 01:20:55,000 --> 01:20:57,920 PARTNERSHIPS ACROSS NIH FOR 2163 01:20:57,920 --> 01:20:59,760 MULTI-SYSTEM DISORDERS AS I JUST 2164 01:20:59,760 --> 01:21:01,720 MINGED AND WE ARE OFTEN -- 2165 01:21:01,720 --> 01:21:03,520 MENTIONED AND WE ARE SOLE 2166 01:21:03,520 --> 01:21:05,160 SUPPORTERS IN LOW PREVALENCE 2167 01:21:05,160 --> 01:21:06,760 DISEASE, AND WE USE -- IT USES 2168 01:21:06,760 --> 01:21:09,160 AND ADVANCES MANY DISEASE AT 2169 01:21:09,160 --> 01:21:13,040 TIME APPROACH AND HAS SINCE DAY 2170 01:21:13,040 --> 01:21:13,520 ONE. 2171 01:21:13,520 --> 01:21:15,920 SUCCESS IS MADE IN ADDRESSING 2172 01:21:15,920 --> 01:21:17,560 CONSORTIA SPECIFIC GAPS IN 2173 01:21:17,560 --> 01:21:19,320 CLINICAL TRIAL READINESS 2174 01:21:19,320 --> 01:21:21,840 PUBLICATIONS INITIATION OF 2175 01:21:21,840 --> 01:21:22,760 LONGITUDINAL OBSERVATIONAL 2176 01:21:22,760 --> 01:21:24,440 NATURAL HISTORY STUDIES. 2177 01:21:24,440 --> 01:21:27,280 NUMBERS OF PATIENTS INCLUDED AND 2178 01:21:27,280 --> 01:21:28,920 PARTICIPATION OF CLINICAL SITES 2179 01:21:28,920 --> 01:21:30,560 IN THE RESEARCH AND THE NUMBER 2180 01:21:30,560 --> 01:21:32,480 OF TYPES OF PILOT STUDIES PEOPLE 2181 01:21:32,480 --> 01:21:34,360 WHO COME INTO THE NETWORK OR 2182 01:21:34,360 --> 01:21:35,920 HAVE IN THE PAST COME IN AT 2183 01:21:35,920 --> 01:21:37,480 DIFFERENT PHASES. 2184 01:21:37,480 --> 01:21:40,480 SOME DON'T HAVE A LOT OF GROUND 2185 01:21:40,480 --> 01:21:42,480 WORK DONE SO THEY ARE STARTING 2186 01:21:42,480 --> 01:21:43,160 AT THE VERY BEGINNING. 2187 01:21:43,160 --> 01:21:44,920 OTHERS COME IN MIDSTREAM, WHERE 2188 01:21:44,920 --> 01:21:47,760 THEY ARE REALLY LOOKING FOR 2189 01:21:47,760 --> 01:21:49,280 BIOMARKERS, AND DEVELOPING NEW 2190 01:21:49,280 --> 01:21:51,520 TOOLS AND SOME ARE READY TO HIT 2191 01:21:51,520 --> 01:21:52,920 THE GROUND RUNNING WITH CLINICAL 2192 01:21:52,920 --> 01:21:53,600 TRIALS. 2193 01:21:53,600 --> 01:21:55,360 SO WE REALLY HAVE A DIVERSE 2194 01:21:55,360 --> 01:22:00,920 GROUP OF NEEDS IN THIS PROGRAM. 2195 01:22:00,920 --> 01:22:03,160 IN SUMMARY, THE FOCUS OF THE 2196 01:22:03,160 --> 01:22:05,360 RDCRN IS TO ADDRESS UNMET 2197 01:22:05,360 --> 01:22:06,360 CLINICAL TRIAL READINESS NEEDS 2198 01:22:06,360 --> 01:22:08,200 THAT MOVE THE FIELD FORWARD FROM 2199 01:22:08,200 --> 01:22:09,640 ITS CURRENT STATE TO 2200 01:22:09,640 --> 01:22:11,320 ESTABLISHMENT AND SUPPORT OF 2201 01:22:11,320 --> 01:22:13,760 CONSORTIA FOR RARE DISEASES. 2202 01:22:13,760 --> 01:22:15,760 FOR CONSORTIA TO ADVANCE 2203 01:22:15,760 --> 01:22:17,400 KNOWLEDGE OF THEIR OWN 2204 01:22:17,400 --> 01:22:18,520 INDIVIDUAL GOALS, AND TO 2205 01:22:18,520 --> 01:22:20,280 LEVERAGE KNOWLEDGE AND RESOURCES 2206 01:22:20,280 --> 01:22:21,600 AVAILABLE WITHIN THE NETWORK AND 2207 01:22:21,600 --> 01:22:23,920 THAT IS ONE THING WE HAVE BEEN 2208 01:22:23,920 --> 01:22:25,000 EMPHASIZING IN THIS CYCLE AND 2209 01:22:25,000 --> 01:22:26,560 WANT TO CONTINUE, IS WE CAN 2210 01:22:26,560 --> 01:22:29,520 REALLY LEARN FROM EACH OTHER. 2211 01:22:29,520 --> 01:22:30,680 THIS INITIATIVE IS EXCITING 2212 01:22:30,680 --> 01:22:32,560 BECAUSE IT IS A PLATFORM THAT 2213 01:22:32,560 --> 01:22:34,160 ENABLES FUTURE CLINICAL TRIALS 2214 01:22:34,160 --> 01:22:36,120 TO DRAW FROM RESEARCH CONDUCTED 2215 01:22:36,120 --> 01:22:37,520 WITHIN THE NETWORK TO BE 2216 01:22:37,520 --> 01:22:39,800 CLINICAL TRIAL READY, TO ATTRACT 2217 01:22:39,800 --> 01:22:40,840 OTHER GROUPS THAT WANT TO WORK 2218 01:22:40,840 --> 01:22:44,760 WITH THEM, AND TO PARTNER. 2219 01:22:44,760 --> 01:22:48,560 NEXT SLIDE PLEASE. 2220 01:22:48,560 --> 01:22:50,000 SO WE ARE GOING TO TAKE SOME 2221 01:22:50,000 --> 01:22:51,760 TIME FOR DISCUSSION, SO THE 2222 01:22:51,760 --> 01:22:53,960 GENERAL QUESTIONS WE HAVE FOR 2223 01:22:53,960 --> 01:22:55,400 COUNCIL ARE WHAT MIGHT PROVE 2224 01:22:55,400 --> 01:22:56,960 THIS INITIATIVE, WHAT ARE THE 2225 01:22:56,960 --> 01:22:57,680 KEY POINTS? 2226 01:22:57,680 --> 01:22:59,680 BUT WHAT WE REALLY LAKE TO ASK 2227 01:22:59,680 --> 01:23:01,000 COUNCIL IS, ARE THERE OTHER 2228 01:23:01,000 --> 01:23:04,080 AREAS OF STUDY OR -- THAT THE 2229 01:23:04,080 --> 01:23:05,760 RDCRN CONSIDER TO HELP ADVANCE 2230 01:23:05,760 --> 01:23:07,880 RARE DISEASE RESEARCH FIELD? 2231 01:23:07,880 --> 01:23:09,440 AND HOW DO YOU RECOMMEND 2232 01:23:09,440 --> 01:23:11,040 ENHANCING COLLABORATION WITH 2233 01:23:11,040 --> 01:23:18,280 OTHER NCATS PROGRAMS? 2234 01:23:18,280 --> 01:23:19,160 OPEN FOR QUESTIONS. 2235 01:23:19,160 --> 01:23:21,240 >> BEFORE WE OPEN UP I WANT TO 2236 01:23:21,240 --> 01:23:23,000 GIVE OPPORTUNITY FOR BECKY AND 2237 01:23:23,000 --> 01:23:24,720 ANNIE AND MARSHALL THE CHIME IN. 2238 01:23:24,720 --> 01:23:26,040 SO BECKY, DO YOU WANT TO GO 2239 01:23:26,040 --> 01:23:30,160 FIRST? 2240 01:23:30,160 --> 01:23:31,640 >> BE HAPPY TO. 2241 01:23:31,640 --> 01:23:33,560 THANK YOU FOR THAT OVERVIEW OF 2242 01:23:33,560 --> 01:23:35,600 THIS CONCEPT AND I'M GOING TO 2243 01:23:35,600 --> 01:23:38,160 REITERATE THE SAME ISSUES THAT I 2244 01:23:38,160 --> 01:23:41,120 DID BEFORE, THAT ACTUAL HISTORY 2245 01:23:41,120 --> 01:23:46,840 STUDIES HAVE REALLY LAID OUT NOT 2246 01:23:46,840 --> 01:23:48,760 ONLY KEY ISSUES IN TERMS OF 2247 01:23:48,760 --> 01:23:51,720 EARLY DIAGNOSIS AND TREATMENT 2248 01:23:51,720 --> 01:23:55,120 OPTIONS, BUT I THINK WHAT THE 2249 01:23:55,120 --> 01:23:56,920 LATE RAMIFICATIONS ARE AND 2250 01:23:56,920 --> 01:24:00,520 INCLUDING THOSE ISSUES 2251 01:24:00,520 --> 01:24:01,880 SURROUNDING KIND OF INTEGRATION 2252 01:24:01,880 --> 01:24:05,160 OF HEALTHCARE AND SUPPORT AND 2253 01:24:05,160 --> 01:24:08,040 ACTUALLY MORE THE WHOLE AREA OF 2254 01:24:08,040 --> 01:24:09,360 SOCIAL SUPPORT. 2255 01:24:09,360 --> 01:24:11,560 I THINK THAT THIS WILL BE A REAL 2256 01:24:11,560 --> 01:24:13,360 OPPORTUNITY FOR NCATS TO THINK 2257 01:24:13,360 --> 01:24:15,120 BROADLY ACROSS AN ENTIRE 2258 01:24:15,120 --> 01:24:17,880 TRANSLATIONAL SPECTRUM TO BE 2259 01:24:17,880 --> 01:24:20,400 ABLE TO ADDRESS THAT AND BUILD 2260 01:24:20,400 --> 01:24:23,160 ADDITIONAL PILOT STUDIES OR 2261 01:24:23,160 --> 01:24:26,320 AREAS IN THAT WHICH ARE SO 2262 01:24:26,320 --> 01:24:28,960 CRITICAL FOR PEOPLE AND AS A 2263 01:24:28,960 --> 01:24:30,680 PARENT OF A YOUNG ADULT NOW, 2264 01:24:30,680 --> 01:24:35,160 WITH A RARE DISEASE, 2265 01:24:35,160 --> 01:24:36,160 ENDOCRINOLOGIST WHO TAKES CARE 2266 01:24:36,160 --> 01:24:38,800 OF PATIENTS WHO ARE ADULTED WITH 2267 01:24:38,800 --> 01:24:40,400 RARE METABOLIC DISEASE, THIS IS 2268 01:24:40,400 --> 01:24:44,000 THE RECURRING THEME, THAT WE 2269 01:24:44,000 --> 01:24:46,040 ADDRESS ON A REGULAR BASIS. 2270 01:24:46,040 --> 01:24:48,520 I THINK MY SECOND ISSUE FOR YOU 2271 01:24:48,520 --> 01:24:51,160 TO THINK ABOUT AND GOES BACK TO 2272 01:24:51,160 --> 01:24:54,440 SOME OF THE COMMENTS MADE 2273 01:24:54,440 --> 01:24:56,560 YESTERDAY, BUT IS REALLY DATA 2274 01:24:56,560 --> 01:24:57,640 EQUITY AND ACCESSIBILITY. 2275 01:24:57,640 --> 01:25:00,440 NOT SURE WHETHER IT REALLY SITS 2276 01:25:00,440 --> 01:25:01,880 HERE OR THE COORDINATING CENTER 2277 01:25:01,880 --> 01:25:04,200 SO I MAYBE REDUNDANT IN SAYING 2278 01:25:04,200 --> 01:25:05,440 IN THE COORDINATING CENTER SO 2279 01:25:05,440 --> 01:25:09,080 FIRST OF ALL, EACH OF -- EACH 2280 01:25:09,080 --> 01:25:10,840 INDIVIDUAL WHO PARTICIPATE IN 2281 01:25:10,840 --> 01:25:12,560 THESE RARE DISEASE I WOULD SAY 2282 01:25:12,560 --> 01:25:14,000 EACH INDIVIDUAL WHO DOE NAITS 2283 01:25:14,000 --> 01:25:17,240 THEIR DATA, SHOULD HAVE THE 2284 01:25:17,240 --> 01:25:19,600 RIGHT TO HAVE B EQUAL ACCESS TO 2285 01:25:19,600 --> 01:25:21,160 INFORMATION IN ORDER TO BE NOT 2286 01:25:21,160 --> 01:25:23,760 ONLY TO UNDERSTAND THINGS 2287 01:25:23,760 --> 01:25:25,200 DIRECTLY RELEVANT TO THEM BUT 2288 01:25:25,200 --> 01:25:26,520 THINGS THAT ARE RELEVANT TO 2289 01:25:26,520 --> 01:25:30,920 PEOPLE THAT ARE LIKE THEM, TO B TO BE 2290 01:25:30,920 --> 01:25:33,360 ABLE GET INSIGHT TO EMERGING. 2291 01:25:33,360 --> 01:25:35,040 SO I ENCOURAGE WHEN YOU THINK 2292 01:25:35,040 --> 01:25:36,240 ABOUT DATA YOU THINK ABOUT THE 2293 01:25:36,240 --> 01:25:40,360 ACCESSIBILITY AND EQUITY FOR 2294 01:25:40,360 --> 01:25:41,440 EVERYONE, SHOULD BE STAKEHOLDERS 2295 01:25:41,440 --> 01:25:42,880 AT THE TABLE, THAT INCLUDES 2296 01:25:42,880 --> 01:25:49,320 FAMILY MEMBERS, AND PATIENTS ALL 2297 01:25:49,320 --> 01:25:51,360 THE WAY OUT THE CLINICIANS AND 2298 01:25:51,360 --> 01:25:52,960 BROADER SCIENTIFIC COMMUNITY 2299 01:25:52,960 --> 01:25:57,360 HELP TO MOVE THESE ISSUES 2300 01:25:57,360 --> 01:25:58,160 FORWARD TOGETHER. 2301 01:25:58,160 --> 01:26:03,560 I GUESS MY VERY LAST SET OF 2302 01:26:03,560 --> 01:26:06,320 ISSUES ARE SEEMS TO BE AWARE OR 2303 01:26:06,320 --> 01:26:10,160 OF, WE HEARD SO MUCH IN THE LAST 2304 01:26:10,160 --> 01:26:11,280 PRESENTATION ABOUT NEW WAY OF 2305 01:26:11,280 --> 01:26:14,120 ACTUALLY THINKING ACROSS 2306 01:26:14,120 --> 01:26:17,960 PATHWAYS OR BIOMOLECULAR SHARED 2307 01:26:17,960 --> 01:26:20,880 ISSUES, AND YET WHAT I DON'T SEE 2308 01:26:20,880 --> 01:26:24,160 IN YOUR CONCEPT IS WHETHER THAT 2309 01:26:24,160 --> 01:26:25,920 IS GOING TO BE FOUGHT IN 2310 01:26:25,920 --> 01:26:28,160 CENTRAL, THINKING FOR EXAMPLE 2311 01:26:28,160 --> 01:26:29,920 GROUPING THINGS NOT BY ORGAN 2312 01:26:29,920 --> 01:26:31,960 SYSTEMS BY BRITTLE BONE DISEASE 2313 01:26:31,960 --> 01:26:34,120 OR RARE BONE DISEASE, BUT IN 2314 01:26:34,120 --> 01:26:37,160 FACT BY SOME OF THE THINGS WE 2315 01:26:37,160 --> 01:26:38,120 KNOW ABOUT THAT WE MIGHT BE ABLE 2316 01:26:38,120 --> 01:26:40,160 TO ACTUALLY FACILITATE 2317 01:26:40,160 --> 01:26:41,440 ADDITIONAL STUDIES. 2318 01:26:41,440 --> 01:26:44,120 SO ENCOURAGE YOU TO BROADEN THAT 2319 01:26:44,120 --> 01:26:48,920 AND ACTUALLY ASK BECAUSE THAT 2320 01:26:48,920 --> 01:26:50,080 WILL MOVE FORWARD UNDERSTANDING 2321 01:26:50,080 --> 01:26:51,800 AND REALLY MUCH MORE 2322 01:26:51,800 --> 01:26:56,160 EFFECTIVENESS AROUND DIAGNOSIS 2323 01:26:56,160 --> 01:26:57,560 AS WELL AS THERAPEUTICS 2324 01:26:57,560 --> 01:27:01,800 DEVELOPMENT. 2325 01:27:01,800 --> 01:27:04,760 >> THANK YOU, BECK KI. 2326 01:27:04,760 --> 01:27:06,280 TIINA, ANY QUICK QUESTIONS TO 2327 01:27:06,280 --> 01:27:07,960 THOSE? 2328 01:27:07,960 --> 01:27:11,520 >> I CAN COMMENT QUICKLY THAT 2329 01:27:11,520 --> 01:27:13,320 THE DATA EQUITY WILL TOUCH ON 2330 01:27:13,320 --> 01:27:16,160 THAT, IN THE NEXT GROUP AND TALK 2331 01:27:16,160 --> 01:27:17,240 ABOUT IT BECAUSE THAT IS 2332 01:27:17,240 --> 01:27:17,920 SOMETHING WE THINK ABOUT 2333 01:27:17,920 --> 01:27:28,040 SERIOUSLY. 2334 01:27:28,040 --> 01:27:29,480 WE THINK OF A BROAD DEFINITION 2335 01:27:29,480 --> 01:27:31,120 WITH THREE RARE DISEASES, IN THE 2336 01:27:31,120 --> 01:27:32,600 LAST CYCLE WE HAD THE DEFINITION 2337 01:27:32,600 --> 01:27:35,040 OF IF YOU HAVE THREE DISEASES OR 2338 01:27:35,040 --> 01:27:36,160 DISORDERS, IF YOU ARE LOOKING AT 2339 01:27:36,160 --> 01:27:37,560 A SIMILAR PATHWAY MANY THE 2340 01:27:37,560 --> 01:27:40,520 DISEASE THAT FOLLOW ON THE 2341 01:27:40,520 --> 01:27:43,760 PATHWAY, WE HAVE A BROAD 2342 01:27:43,760 --> 01:27:48,760 DEFINITION HOW GROUPS COME IN 2343 01:27:48,760 --> 01:27:50,560 AND CLUSTER WHAT THEY ARE 2344 01:27:50,560 --> 01:27:51,240 STUDYING AND HOW. 2345 01:27:51,240 --> 01:27:52,440 THANK YOU FOR BRINGING THAT UP, 2346 01:27:52,440 --> 01:27:53,920 THAT IS A GOOD POINT AND 2347 01:27:53,920 --> 01:27:55,480 SOMETHING WE TAKE SERIOUSLY. 2348 01:27:55,480 --> 01:27:57,160 AND ON OCCASION, WE HAVE A 2349 01:27:57,160 --> 01:28:02,600 CHALLENGE WITH THAT BECAUSE 2350 01:28:02,600 --> 01:28:05,240 SOMETIMES REVIEWERS LOOK AT IT 2351 01:28:05,240 --> 01:28:08,640 AT THREE ORGAN SYSTEMS AT A 2352 01:28:08,640 --> 01:28:10,440 TIME, THREE DISEASE CLASSES AT A 2353 01:28:10,440 --> 01:28:11,000 TIME. 2354 01:28:11,000 --> 01:28:13,560 AND TRYING TO BRING ACROSS A NEW 2355 01:28:13,560 --> 01:28:19,840 WAY OF THINKING, WORKING TO 2356 01:28:19,840 --> 01:28:20,920 ENCOURAGE OTHERS TO THINK THE 2357 01:28:20,920 --> 01:28:21,400 WAY WE THINK. 2358 01:28:21,400 --> 01:28:26,840 THANK YOU. 2359 01:28:26,840 --> 01:28:28,040 >> THANK YOU VERY MUCH. 2360 01:28:28,040 --> 01:28:31,360 >> FULL DISCLOSURE, WHEN I -- 2361 01:28:31,360 --> 01:28:34,080 OFFICE OF POLICY PROGRAM FOR THE 2362 01:28:34,080 --> 01:28:36,640 MUSCULAR DYSTROPHY ASSOCIATION, 2363 01:28:36,640 --> 01:28:39,480 THIS IS MY FIRST INITIATION, I 2364 01:28:39,480 --> 01:28:47,160 SERVED AS THE RAP TO THREE 2365 01:28:47,160 --> 01:28:53,160 CONSORTIA, (INAUDIBLE) SO THIS 2366 01:28:53,160 --> 01:28:54,400 IS ONE OF MY FAVORITE PROGRAMS 2367 01:28:54,400 --> 01:28:56,920 AND ONE THAT HAD A LASTING 2368 01:28:56,920 --> 01:28:58,400 IMPACT IN THE COMMUNITY. 2369 01:28:58,400 --> 01:29:06,480 MY FIRST COMMENT IS IF I MISSED 2370 01:29:06,480 --> 01:29:08,360 IT I APOLOGIZE BUT I THINK THE 2371 01:29:08,360 --> 01:29:10,800 PROGRAM IS SCREAMING FOR INFO 2372 01:29:10,800 --> 01:29:11,560 GRAPHICS. 2373 01:29:11,560 --> 01:29:13,480 AS ANNE WAS GOING THROUGH AND 2374 01:29:13,480 --> 01:29:15,440 TIINA TALKING ABOUT TELLING THE 2375 01:29:15,440 --> 01:29:20,160 STORY OF THE CONSORTIA, AND YOU 2376 01:29:20,160 --> 01:29:22,480 WENT THROUGH METRICS, NUMBER OF 2377 01:29:22,480 --> 01:29:25,840 ICs INVOLVE, NUMBER OF PAG. 2378 01:29:25,840 --> 01:29:27,680 ACADEMICICS DISEASES COVERED, 2379 01:29:27,680 --> 01:29:29,120 NUMBER OF CLINICAL TRIALS, 2380 01:29:29,120 --> 01:29:31,200 THERAPIES, ET CETERA, ET CETERA, 2381 01:29:31,200 --> 01:29:32,960 I MIGHT SUGGEST CUPFUL O OVER 2382 01:29:32,960 --> 01:29:34,960 MEASURES TO TIE TO OTHER THINGS 2383 01:29:34,960 --> 01:29:39,200 NCATS WORK OBS THAT MAKE MORE BY 2384 01:29:39,200 --> 01:29:41,600 NUMBERS WHAT CONSORTIA 2385 01:29:41,600 --> 01:29:43,880 ACCOMPLISHED, BOTH GNASH 2386 01:29:43,880 --> 01:29:46,360 MILLIONLY AND GLOBALLY TO THE 2387 01:29:46,360 --> 01:29:47,400 POINT MADE EARLIER CONVERSATIONS 2388 01:29:47,400 --> 01:29:49,200 BECAUSE THOSE PARTNERS ARE 2389 01:29:49,200 --> 01:29:52,000 WORKING ACROSS GLOBE AS WELL. 2390 01:29:52,000 --> 01:29:53,600 BUT CAN WE LOOK AT METRICS 2391 01:29:53,600 --> 01:29:55,560 AROUND SHORTENING DIAGNOSTIC 2392 01:29:55,560 --> 01:29:56,760 ODYSSEY, WHAT THOSE EFFORTS HAVE 2393 01:29:56,760 --> 01:30:00,360 DONE AROUND THAT, ANY CONDITIONS 2394 01:30:00,360 --> 01:30:01,840 ADDED AS A RESULT OF THE EFFORTS 2395 01:30:01,840 --> 01:30:03,960 OF THESE GROUPS. AND THEN HAVE 2396 01:30:03,960 --> 01:30:06,920 ANY OF THE RESOURCES THAT HAVE 2397 01:30:06,920 --> 01:30:09,880 COME FROM THE CONSORTIA BEEN 2398 01:30:09,880 --> 01:30:11,360 LEVERAGED FOR PATIENT FOCUSED 2399 01:30:11,360 --> 01:30:13,680 DRUG DEVELOPMENT WORKSHOPS, 2400 01:30:13,680 --> 01:30:15,560 ENGAGEMENTS WITH FDA OR CMS. 2401 01:30:15,560 --> 01:30:17,360 THIS MIGHT BE SOME OTHER METRICS 2402 01:30:17,360 --> 01:30:18,760 BUT I WOULD START DIGGING INTO 2403 01:30:18,760 --> 01:30:22,000 AND LOOKING AT AND COUNTING. 2404 01:30:22,000 --> 01:30:23,440 MY OTHER COMMENT IS JUST MAKE 2405 01:30:23,440 --> 01:30:26,640 MAYBE A QUESTION, WHEN YOU ARE 2406 01:30:26,640 --> 01:30:28,960 REALLY SOLICITING AND 2407 01:30:28,960 --> 01:30:31,000 DETERMINING WHICH CONSORTIA, IT 2408 01:30:31,000 --> 01:30:32,760 IS A COMPETITIVE PROCESS, I KNOW 2409 01:30:32,760 --> 01:30:35,560 COMMUNITIES ARE EAGER TO BE 2410 01:30:35,560 --> 01:30:36,680 AMONG THEM, HOW DO YOU THINK 2411 01:30:36,680 --> 01:30:38,920 ABOUT THE ULTRA RARE 2412 01:30:38,920 --> 01:30:43,280 COMMUNITIES, AND IS THERE ANY 2413 01:30:43,280 --> 01:30:44,560 PERCENTAGE OF CONSORTIA TO SEE 2414 01:30:44,560 --> 01:30:46,200 IN THE ULTRA RARE COMMUNITY OR 2415 01:30:46,200 --> 01:30:49,280 IS THAT SOMETHING TO THINK ABOUT 2416 01:30:49,280 --> 01:30:51,880 SO MAYBE WE CAN PUT A PIN IN 2417 01:30:51,880 --> 01:30:54,760 THAT. 2418 01:30:54,760 --> 01:30:56,360 WE SEE UNDER-REPRESENTED, 2419 01:30:56,360 --> 01:30:58,040 UNDERFUNDED. 2420 01:30:58,040 --> 01:30:58,640 UNDERRESOURCEED. 2421 01:30:58,640 --> 01:31:03,360 THE LAST THING AS SOMETHING WHO 2422 01:31:03,360 --> 01:31:05,200 HAS BEEN A PAG MEMBER I WAS 2423 01:31:05,200 --> 01:31:07,840 STRUCK BY NUMBER OF PAG, I THINK 2424 01:31:07,840 --> 01:31:12,840 IT WAS 166 PA DWRKS FOR 20 2425 01:31:12,840 --> 01:31:16,960 CONSORTIAS, -- PAGS FOR 20 2426 01:31:16,960 --> 01:31:17,560 CONSORTIAS. 2427 01:31:17,560 --> 01:31:18,600 ALL ARE NOT EQUAL. 2428 01:31:18,600 --> 01:31:20,160 YOU HAD A LOT OF RESOURCES TO 2429 01:31:20,160 --> 01:31:21,600 BRING TO BARE AND SOME WITHIN 2430 01:31:21,600 --> 01:31:23,920 THE CONSORTIA, I WORK WITH THAT 2431 01:31:23,920 --> 01:31:25,880 REALLY WEREN'T SURE HOW TO 2432 01:31:25,880 --> 01:31:27,280 ENGAGE AND WHAT TO BRING TO THE 2433 01:31:27,280 --> 01:31:31,240 TABLE. 2434 01:31:31,240 --> 01:31:32,480 SO ONE OF THE COMMENTS, WE ASK A 2435 01:31:32,480 --> 01:31:34,160 LOT OF PATIENT ADVOCACY PARTNERS 2436 01:31:34,160 --> 01:31:38,040 WHEN WE ASK THEM TO BE PART OF 2437 01:31:38,040 --> 01:31:38,760 SOMETHING. 2438 01:31:38,760 --> 01:31:40,320 TAKES TIME BANDWIDTH PERSONNEL 2439 01:31:40,320 --> 01:31:41,760 WHICH IS TAKING AWAY FROM THE 2440 01:31:41,760 --> 01:31:42,760 BOTTOM LINE OF AN ORGANIZATION 2441 01:31:42,760 --> 01:31:43,960 AND WE ARE NOT PROVIDING FUNDING 2442 01:31:43,960 --> 01:31:46,200 FOR THAT. 2443 01:31:46,200 --> 01:31:48,320 MANY TIMES PEOPLE BRING DATA TO 2444 01:31:48,320 --> 01:31:50,880 US WE CAN TALK ABOUT AND BECKY 2445 01:31:50,880 --> 01:31:52,520 TALKED ABOUT, WILL THEY ARE 2446 01:31:52,520 --> 01:31:54,040 SENDING OUT DISTRIBUTIONS TO 2447 01:31:54,040 --> 01:31:56,000 THEIR MAILING LIST, THEY ARE DO 2448 01:31:56,000 --> 01:31:58,240 A LOT TO BE GOOD PARTNERS, ONE 2449 01:31:58,240 --> 01:31:59,760 QUESTION I WOULD ASK IS HOW 2450 01:31:59,760 --> 01:32:02,680 INTENTIONAL ARE WE BEING ABOUT 2451 01:32:02,680 --> 01:32:06,400 WHAT BENEFIT THE PA GRKs 2452 01:32:06,400 --> 01:32:08,200 DERIVE, BEYOND BENEFIT TO 2453 01:32:08,200 --> 01:32:10,400 COMMUNITY, BUT ARE THE PAGs 2454 01:32:10,400 --> 01:32:13,680 THEMSELVES COMING FROM THIS 2455 01:32:13,680 --> 01:32:17,360 STRONGER BETTER, MORE EQUIPPED 2456 01:32:17,360 --> 01:32:19,920 TO ENGAGE WITHIN THIS 2457 01:32:19,920 --> 01:32:22,160 ENVIRONMENT. 2458 01:32:22,160 --> 01:32:23,480 NCATS IS WELL-POSITIONED TO DO 2459 01:32:23,480 --> 01:32:25,360 MENTORING HERE WITH THE PAGs 2460 01:32:25,360 --> 01:32:27,440 THAT COME IN AND NOT LEAVE TO 2461 01:32:27,440 --> 01:32:28,760 PIs OF THE CONSORTIA TO DO 2462 01:32:28,760 --> 01:32:29,280 THAT. 2463 01:32:29,280 --> 01:32:31,560 SO THE NCATS TOOL KIT IS A GOOD 2464 01:32:31,560 --> 01:32:32,400 PLACE TO START. 2465 01:32:32,400 --> 01:32:35,160 AND TO MAYBE EVEN HAVE ALMOST A 2466 01:32:35,160 --> 01:32:36,640 AND A HALF GAITION OF THE 2467 01:32:36,640 --> 01:32:38,480 RESOURCES AND -- NAVIGATION AND 2468 01:32:38,480 --> 01:32:39,840 RESOURCES AND HAVING PEOPLE 2469 01:32:39,840 --> 01:32:40,920 IDENTIFY WHERE THEY ARE, TO 2470 01:32:40,920 --> 01:32:42,520 IDENTIFY WHAT CAPACITIES AND 2471 01:32:42,520 --> 01:32:44,360 RESOURCES THEY HAVE TO BRING TO 2472 01:32:44,360 --> 01:32:46,200 BEAR ON THE AIMS OF THAT 2473 01:32:46,200 --> 01:32:49,320 PARTICULAR CONSORTIA AND 2474 01:32:49,320 --> 01:32:53,160 IDENTIFICATION OF ALL THE PAGES, 2475 01:32:53,160 --> 01:32:56,000 I BELIEVE THERE WAS 15 WORKING 2476 01:32:56,000 --> 01:32:58,080 WITHIN IT WHO WAS GOING TO BRING 2477 01:32:58,080 --> 01:33:00,280 WHAT, COULD THERE BE CONSORTIA 2478 01:33:00,280 --> 01:33:03,280 AMONG PAG MEMBERS AND MAYBE 2479 01:33:03,280 --> 01:33:05,600 REDUCE SOME OF THE COMPETITION 2480 01:33:05,600 --> 01:33:07,360 AMONG THEM AND BRING TOGETHER 2481 01:33:07,360 --> 01:33:08,400 PAGES IN SOME WAYS. 2482 01:33:08,400 --> 01:33:10,600 MAYBE INTENTIONAL WORK ON WHAT 2483 01:33:10,600 --> 01:33:15,880 WE DO FOR THE PAGs PARTNER. 2484 01:33:15,880 --> 01:33:17,520 AND THEN WHAT THEY COME AWAY 2485 01:33:17,520 --> 01:33:20,400 WITH SO THAT SOMETHING TO THINK 2486 01:33:20,400 --> 01:33:22,360 ABOUT BECAUSE THERE'S SO MUCH 2487 01:33:22,360 --> 01:33:24,280 ASKED OF OUR PATIENT ADVOCACY 2488 01:33:24,280 --> 01:33:25,920 GROUPS TODAY IN THIS DAY AND 2489 01:33:25,920 --> 01:33:27,160 AGE. 2490 01:33:27,160 --> 01:33:29,280 AND SEEMS TO ME NCATS IS A 2491 01:33:29,280 --> 01:33:30,520 WONDERFUL PLACE FOR PEOPLE TO 2492 01:33:30,520 --> 01:33:33,080 COME STRONGER BETTER, AND WILL 2493 01:33:33,080 --> 01:33:35,160 BE FOREVER SPOKESPEOPLE. 2494 01:33:35,160 --> 01:33:39,480 BECAUSE OF THIS RELATIONSHIP. 2495 01:33:39,480 --> 01:33:40,760 >> FANTASTIC IDEA. 2496 01:33:40,760 --> 01:33:40,920 GREAT. 2497 01:33:40,920 --> 01:33:41,320 THANK YOU. 2498 01:33:41,320 --> 01:33:44,800 >> THOSE ARE GREAT IDEAS AND I 2499 01:33:44,800 --> 01:33:45,800 ESPECIALLY BECAUSE ONE OF THE 2500 01:33:45,800 --> 01:33:51,920 IDEAS YOU HAD WAS WITH THE INFO 2501 01:33:51,920 --> 01:33:53,480 GRAPHICS, RIGHT NOW WITH THE 2502 01:33:53,480 --> 01:33:55,600 LIFE CYCLE WE MADE CHANGES WITH 2503 01:33:55,600 --> 01:33:57,920 RDCRN AND THE DNCC AND WITH THE 2504 01:33:57,920 --> 01:34:00,640 PATIENT ADVOCACY GROUPS IN HOW 2505 01:34:00,640 --> 01:34:01,840 WE APPROACH THINGS HOW WE ARE 2506 01:34:01,840 --> 01:34:02,760 DOING THINGS. 2507 01:34:02,760 --> 01:34:04,440 SO THE INFO GRAPHICS IS GREAT 2508 01:34:04,440 --> 01:34:07,920 BECAUSE THEY ARE REDOING WEB 2509 01:34:07,920 --> 01:34:09,200 SITES AND THEY ARE PROCESS OF 2510 01:34:09,200 --> 01:34:10,880 DOING THAT NOW SO SHARING IDEAS 2511 01:34:10,880 --> 01:34:12,360 WITH THEM NOW CAN GET 2512 01:34:12,360 --> 01:34:13,160 IMPLEMENTED EARLIER THAN THE 2513 01:34:13,160 --> 01:34:13,800 NEXT CYCLE. 2514 01:34:13,800 --> 01:34:17,520 SO WE CAN GO RIGHT INTO THAT. 2515 01:34:17,520 --> 01:34:20,080 I LIKE THE SHOUT OUT FOR HOW WE 2516 01:34:20,080 --> 01:34:23,240 HELP GET CONDITIONS ON THE -- 2517 01:34:23,240 --> 01:34:29,120 NEWBORN SCREENING FAN. 2518 01:34:29,120 --> 01:34:30,440 WE MADE DIFFERENCES IN HOW WE 2519 01:34:30,440 --> 01:34:34,600 ARE WORKING WITH PATIENT AD VO 2520 01:34:34,600 --> 01:34:35,200 ADVOCACY GROUPS. 2521 01:34:35,200 --> 01:34:37,600 SO THEY ARE BEING SUPPORTED BY 2522 01:34:37,600 --> 01:34:39,160 DATA MANAGEMENT AND COORDINATING 2523 01:34:39,160 --> 01:34:42,040 CENTER, THEY HAVE A TEAM THERE, 2524 01:34:42,040 --> 01:34:43,120 THEY HAVE A STEERING COMMITTEE 2525 01:34:43,120 --> 01:34:44,720 OF THAT ARE OWN WHERE EACH 2526 01:34:44,720 --> 01:34:49,120 PATIENT ADVOCACY GROUPS PIKS ONE 2527 01:34:49,120 --> 01:34:50,440 REP ON THE STEERING COMMITTEE 2528 01:34:50,440 --> 01:34:53,920 AND THEY ROTATE OFF EACH YEAR OR 2529 01:34:53,920 --> 01:34:55,920 YEAR AND A HALF SO DON'T 2530 01:34:55,920 --> 01:34:56,720 OVERBURDEN ONE PERSON. 2531 01:34:56,720 --> 01:35:01,240 WHENEVER WE ARE HAVING MEETINGS 2532 01:35:01,240 --> 01:35:02,440 TRYING TO FIGURE WHAT PEOPLE 2533 01:35:02,440 --> 01:35:04,320 WANT WE ASK THEM WHAT DO YOU 2534 01:35:04,320 --> 01:35:06,120 WANT, WHAT DO YOU WANT TO LEARN 2535 01:35:06,120 --> 01:35:07,640 FROM, WE KEEP FOCUS ON RESEARCH 2536 01:35:07,640 --> 01:35:11,360 BUT WE SAY WE CAN'T DO 2537 01:35:11,360 --> 01:35:12,600 EVERYTHING FOR YOU BUT IN THIS 2538 01:35:12,600 --> 01:35:13,840 -- WITHIN THE SCOPE OF RESEARCH, 2539 01:35:13,840 --> 01:35:19,920 HOW CAN WE WORK WITH YOU. 2540 01:35:19,920 --> 01:35:21,560 PROGRAM ESTABLISH MENTORS FOR 2541 01:35:21,560 --> 01:35:22,360 NEW GROUPS BECAUSE WE RECOGNIZE 2542 01:35:22,360 --> 01:35:23,560 THE FACT THAT YOU MIGHT HAVE 2543 01:35:23,560 --> 01:35:25,160 SOMEONE WHO IS SITTING IN THEIR 2544 01:35:25,160 --> 01:35:28,080 KITCHEN TABLE IN KENTUCKY AND 2545 01:35:28,080 --> 01:35:30,200 THEN YOU HAVE ONES WITH FANCY 2546 01:35:30,200 --> 01:35:31,400 OFFICES IN WASHINGTON D.C. OR 2547 01:35:31,400 --> 01:35:40,480 NEW YORK. 2548 01:35:40,480 --> 01:35:44,240 LETS GROUP AT SAME LEVEL OF 2549 01:35:44,240 --> 01:35:45,560 EXPERIENCE LEARN TOGETHER LEARN 2550 01:35:45,560 --> 01:35:47,920 FROM EACH OTHER, TRYING TO 2551 01:35:47,920 --> 01:35:49,400 ESTABLISH A COMMUNITY WHERE 2552 01:35:49,400 --> 01:35:50,840 PEOPLE CAN LEARN FROM EACH OTHER 2553 01:35:50,840 --> 01:35:52,080 AND MENTOR EACH OTHER. 2554 01:35:52,080 --> 01:35:55,560 BUT IT IS ALSO GREAT, TO BRING 2555 01:35:55,560 --> 01:35:56,600 TOOLS TO THEM, WE BROUGHT THE 2556 01:35:56,600 --> 01:35:58,040 PAD THE TALK TO THEM WHEN YOU 2557 01:35:58,040 --> 01:35:58,440 HAVE QUESTIONS. 2558 01:35:58,440 --> 01:36:01,840 WE DON'T UNDERSTAND THE FDA 2559 01:36:01,840 --> 01:36:03,600 TIINA, LET'S BRING SOMEONE TO 2560 01:36:03,600 --> 01:36:05,360 TABLE SO WE WILL BRING WHOEVER 2561 01:36:05,360 --> 01:36:06,920 THEY ASK FOR AND I FEEL THAT IT 2562 01:36:06,920 --> 01:36:08,520 IS IMPORTANT THAT WHEN SOMEBODY 2563 01:36:08,520 --> 01:36:12,560 -- ESPECIALLY THE PATIENT AD VO 2564 01:36:12,560 --> 01:36:13,600 ADVOCACY GROUP LEAVES A MEETING 2565 01:36:13,600 --> 01:36:14,840 THEY ARE TAKING SOMETHING 2566 01:36:14,840 --> 01:36:17,560 HEADACHE USE AFTER THAT MEETING. 2567 01:36:17,560 --> 01:36:19,920 THANK YOU FOR THAT POINT, ANNIE. 2568 01:36:19,920 --> 01:36:22,320 >> THANK YOU. 2569 01:36:22,320 --> 01:36:23,720 EMPHASIS OF THOSE ACTIVITIES 2570 01:36:23,720 --> 01:36:26,160 JUST TO ANNIE'S POINT OF 2571 01:36:26,160 --> 01:36:30,400 ENSURING THAT WE ARE DOING EVEN 2572 01:36:30,400 --> 01:36:32,600 MORE THINKING ABOUT NAVIGATING 2573 01:36:32,600 --> 01:36:33,720 AND COORDINATING IN THOSE 2574 01:36:33,720 --> 01:36:34,120 THINGS. 2575 01:36:34,120 --> 01:36:35,360 SO THANK YOU. 2576 01:36:35,360 --> 01:36:39,000 PETER I SEE YOUR HAND UP BEFORE 2577 01:36:39,000 --> 01:36:41,440 MARSHALL. 2578 01:36:41,440 --> 01:36:43,360 >> A QUICK COMMENT IN ADDITION 2579 01:36:43,360 --> 01:36:44,280 TO WHAT TIINA SAID. 2580 01:36:44,280 --> 01:36:46,360 HUE FOR BY BRINGING UP THE TOOL 2581 01:36:46,360 --> 01:36:46,760 KIT. 2582 01:36:46,760 --> 01:36:48,480 WE TRIED TO STAY ON TIME TO GIVE 2583 01:36:48,480 --> 01:36:49,920 PEOPLE A CHANCE TO ASK QUESTIONS 2584 01:36:49,920 --> 01:36:51,560 SO WE DIDN'T HIT EVERYTHING WE 2585 01:36:51,560 --> 01:36:56,280 DO IN ORDR BUT TOOL KIT IS 2586 01:36:56,280 --> 01:36:57,040 IMPORTANT PROGRAM. 2587 01:36:57,040 --> 01:37:00,160 FOLLOWING UP ON YOU AND BECKY, 2588 01:37:00,160 --> 01:37:03,800 PATHWAYS AND ULTRA RARE, THOSE 2589 01:37:03,800 --> 01:37:04,920 TWO THINGS COME TOGETHER 2590 01:37:04,920 --> 01:37:08,200 THINKING WAYS TO GROUP DISEASE, 2591 01:37:08,200 --> 01:37:11,920 TIINA ALLUDED TO THIS ALSO THE 2592 01:37:11,920 --> 01:37:13,760 CURRENT ITERATION OF RDCRN WE 2593 01:37:13,760 --> 01:37:16,960 HAVE PATHWAY FOCUSED APPROACHES 2594 01:37:16,960 --> 01:37:18,440 INCLUDING CONGENITAL DISORDERS 2595 01:37:18,440 --> 01:37:20,240 OF GLYCOSYLATION AND I THINK WE 2596 01:37:20,240 --> 01:37:21,360 WILL CONTINUE TO ENCOURAGE 2597 01:37:21,360 --> 01:37:23,200 PEOPLE TO THINK ABOUT 2598 01:37:23,200 --> 01:37:27,040 ALTERNATIVE GROUPINGS THAT SOME 2599 01:37:27,040 --> 01:37:28,320 EXTENT REDEFINE WHAT DISEASE IS 2600 01:37:28,320 --> 01:37:29,720 AND CAN ADDRESS ISSUE HOW WE 2601 01:37:29,720 --> 01:37:34,520 DEAL WITH I WILL TR WITH ULTRA RARE DISORD ER. 2602 01:37:34,520 --> 01:37:35,200 >> MARSHALL. 2603 01:37:35,200 --> 01:37:37,560 GO I HEAD. 2604 01:37:37,560 --> 01:37:40,680 >> I GO BACK WITH RDCRN BEFORE 2605 01:37:40,680 --> 01:37:44,560 IT WAS RDCR IN IN 2000 WHEN 2606 01:37:44,560 --> 01:37:46,760 STEVE WAS IN THE BASEMENT 2607 01:37:46,760 --> 01:37:49,720 LEADING TO CONSORTIUM AND I WAS 2608 01:37:49,720 --> 01:37:51,360 ONE OF THE ORIGINAL PI E FOR ONE 2609 01:37:51,360 --> 01:37:55,240 OF THE FIRST UREA CYCLES 2610 01:37:55,240 --> 01:37:56,680 CONSORTIUM IN 2004 THAT WILL 2611 01:37:56,680 --> 01:37:58,200 FINALLY SUNSET AFTER MANY 2612 01:37:58,200 --> 01:38:00,160 CYCLES. 2613 01:38:00,160 --> 01:38:01,360 I HAVE BEEN INVOLVED IN 2614 01:38:01,360 --> 01:38:03,200 EDUCATION IN THE RDCRN. 2615 01:38:03,200 --> 01:38:04,760 SO THE REASON I SAY THIS IS I 2616 01:38:04,760 --> 01:38:08,760 HAVE A FAIRLY DEEP BASIS OF 2617 01:38:08,760 --> 01:38:09,760 EXPERIENCING THE PROGRAM SINCE 2618 01:38:09,760 --> 01:38:12,120 INCEPTION. 2619 01:38:12,120 --> 01:38:14,560 I THINK LOOKING ACROSS THE NIH 2620 01:38:14,560 --> 01:38:15,640 PORTFOLIO ACROSS ALL CENTERS 2621 01:38:15,640 --> 01:38:17,760 THIS IS ACTUALLY ONE OF THE MORE 2622 01:38:17,760 --> 01:38:19,320 SUCCESSFUL PROGRAMS. 2623 01:38:19,320 --> 01:38:20,680 HOWEVER, THERE ARE SOME THINGS I 2624 01:38:20,680 --> 01:38:22,000 THINK BEDEFINITELY NEED TO THINK 2625 01:38:22,000 --> 01:38:23,240 ABOUT. 2626 01:38:23,240 --> 01:38:26,200 -- WE DEFINITELY IMMEDIATE TO 2627 01:38:26,200 --> 01:38:27,160 THINK ABOUT. 2628 01:38:27,160 --> 01:38:30,440 LEGACY ISSUES AROUND DATA AND 2629 01:38:30,440 --> 01:38:32,120 NETWORKS PUT TOGETHER WITH A LOT 2630 01:38:32,120 --> 01:38:36,360 OF TIME AND TREASURE, THAT THE 2631 01:38:36,360 --> 01:38:38,520 NIH CANNOT FUND FOREVER NOR 2632 01:38:38,520 --> 01:38:40,200 SHOULD IT BUT NOT SURE WE HAVE 2633 01:38:40,200 --> 01:38:41,880 GIVEN THOUGHT HOW THE DATA THAT 2634 01:38:41,880 --> 01:38:43,320 IS COLLECTED PRESERVED MADE 2635 01:38:43,320 --> 01:38:45,160 AVAILABLE SO IT CAN BE CARRIED 2636 01:38:45,160 --> 01:38:48,520 FORWARD AND WHAT EFFORTS CAN WE 2637 01:38:48,520 --> 01:38:51,520 MAKE FOR THOSE CONSORTIUM TO 2638 01:38:51,520 --> 01:38:52,320 HAVING LIFE AFTER. 2639 01:38:52,320 --> 01:38:54,560 BECAUSE WE HAVE GOT 20 ALMOST 2640 01:38:54,560 --> 01:38:57,160 YEARS OF DATA IN THE UREA CYCLE 2641 01:38:57,160 --> 01:38:58,200 AND OTHER GROUPS, THAT IS 2642 01:38:58,200 --> 01:38:59,560 SCRATCHING THE SURFACE, AS WE 2643 01:38:59,560 --> 01:39:00,880 LOOK AT PATIENT WHOSE ARE 2644 01:39:00,880 --> 01:39:04,800 BECOMING ADULTS, PATIENTS 2645 01:39:04,800 --> 01:39:05,680 FURTHER SOME OF THE INTERESTING 2646 01:39:05,680 --> 01:39:07,160 THINGS YOU FIND THE THINGS YOU 2647 01:39:07,160 --> 01:39:10,760 NEED TO KNOW ABOUT DON'T HAPPEN. 2648 01:39:10,760 --> 01:39:11,360 UNTIL AFTER. 2649 01:39:11,360 --> 01:39:12,360 SO I THINK ONE THING I WOULD 2650 01:39:12,360 --> 01:39:15,560 LIKE TO SEE IS A CONCERTED AND 2651 01:39:15,560 --> 01:39:17,680 STANDARDIZED APPROACH TO WHETHER 2652 01:39:17,680 --> 01:39:19,920 WE INVOLVE ONE OF THE OTHER 2653 01:39:19,920 --> 01:39:22,120 NATIONAL PATIENT ORGANIZATIONS 2654 01:39:22,120 --> 01:39:23,760 AND DOES THE DATA BECOME 2655 01:39:23,760 --> 01:39:25,280 CUSTODIAL THERE AND USEFUL 2656 01:39:25,280 --> 01:39:31,000 THROUGH PATIENT A ADVOCACY GROUP, 2657 01:39:31,000 --> 01:39:31,960 NUMBER OF POSSIBILITIES FOR 2658 01:39:31,960 --> 01:39:32,640 THAT. 2659 01:39:32,640 --> 01:39:35,200 DURING MY TIME AS PI WE CARRIED 2660 01:39:35,200 --> 01:39:37,360 OVER TEN YEAR PERIOD LEE DRUGS 2661 01:39:37,360 --> 01:39:40,320 THROUGH FDA APPROVAL. 2662 01:39:40,320 --> 01:39:43,480 WHAT WE FOUND IS THE DATA IN THE 2663 01:39:43,480 --> 01:39:44,600 ACTUAL NATURAL HISTORY REGISTRY 2664 01:39:44,600 --> 01:39:46,360 WAS NOT UP TO FDA STANDARDS SO 2665 01:39:46,360 --> 01:39:49,240 WE HAVE TO CREATE PARALLEL 2666 01:39:49,240 --> 01:39:49,480 PROCESS. 2667 01:39:49,480 --> 01:39:51,280 WE USE OUR NETWORK BUT NOT ABLE 2668 01:39:51,280 --> 01:39:57,160 TO USE THE DATA FROM THE RDCRN. 2669 01:39:57,160 --> 01:40:00,320 I WOULD STRONGLY LOOK AT WAYS 2670 01:40:00,320 --> 01:40:01,960 WHERE WE CAN BRING FATD FOLKS IN 2671 01:40:01,960 --> 01:40:03,440 LOOK AT THE DATA STRUCTURE WE 2672 01:40:03,440 --> 01:40:04,360 BUILT BECAUSE ONE OF THE BEST 2673 01:40:04,360 --> 01:40:05,760 THINGS ABOUT HAVING THESE IS THE 2674 01:40:05,760 --> 01:40:07,000 ABILITY TO BRING NEW TREATMENT. 2675 01:40:07,000 --> 01:40:08,840 WE LEARN ABOUT THE DISEASE. 2676 01:40:08,840 --> 01:40:10,760 WE ABSOLUTELY DO. 2677 01:40:10,760 --> 01:40:14,160 BUT ALSO THIS DATA CAN BE 2678 01:40:14,160 --> 01:40:15,480 LEVERAGED AND PROVIDING GOOD 2679 01:40:15,480 --> 01:40:18,440 DATA STRUCTURE FOR THOSE AND 2680 01:40:18,440 --> 01:40:20,160 LIKE I SAID I KNOW MADE A LOT OF 2681 01:40:20,160 --> 01:40:22,520 EFFORTS THAT ARE -- BUT ONE 2682 01:40:22,520 --> 01:40:24,360 THING AND THIS OVERLAPS TO THE 2683 01:40:24,360 --> 01:40:26,800 NEXT MCC, WHICH IS WHAT CAN WE 2684 01:40:26,800 --> 01:40:29,680 DO AROUND STANDARDIZATION OF 2685 01:40:29,680 --> 01:40:32,480 TERMS, DATA COLLECTION, 2686 01:40:32,480 --> 01:40:35,320 INSTRUMENT TOOLS, WHAT NEW ONES 2687 01:40:35,320 --> 01:40:37,120 NEED TO BE BROUGHT FORWARD PJ 2688 01:40:37,120 --> 01:40:39,200 YOU TALKED ABOUT SOME OF THE 2689 01:40:39,200 --> 01:40:40,440 PROJECTS YOU GOT TO LOOK AT 2690 01:40:40,440 --> 01:40:43,560 DEVELOPING NEW TOOLS, YOU RDCRN 2691 01:40:43,560 --> 01:40:45,720 IS A GREAT PLACE TO DO THAT, YOU 2692 01:40:45,720 --> 01:40:47,360 GOT YOURSELF CAPTIVE AUDIENCE 2693 01:40:47,360 --> 01:40:48,760 FOR DOING SOME OF THOSE THINGS. 2694 01:40:48,760 --> 01:40:50,600 SO I REALLY LIKE TO SEE MORE 2695 01:40:50,600 --> 01:40:52,440 WORK LIKE THAT. 2696 01:40:52,440 --> 01:40:56,200 LAST COMMENT. 2697 01:40:56,200 --> 01:40:57,360 ONE THING WE FOUND IS A 2698 01:40:57,360 --> 01:41:00,840 GEOGRAPHIC COMPONENT TO THE 2699 01:41:00,840 --> 01:41:02,040 RDCRN. 2700 01:41:02,040 --> 01:41:04,240 PATIENTS CLOSE TO ASSISTANTER 2701 01:41:04,240 --> 01:41:05,360 TYPICALLY GOT TO PARTICIPATE. 2702 01:41:05,360 --> 01:41:07,480 IT WAS A TIGHT BURDEN OF 2703 01:41:07,480 --> 01:41:09,800 PARTICIPATION FOR FAMILIES IN 2704 01:41:09,800 --> 01:41:10,800 MORE REMOTE AREAS. 2705 01:41:10,800 --> 01:41:12,120 ONE THING I WOULD LOVE THE SEE 2706 01:41:12,120 --> 01:41:15,640 US DO, THERE IS ACCESS TO EQUITY 2707 01:41:15,640 --> 01:41:16,680 ISSUE HERE TOO. 2708 01:41:16,680 --> 01:41:18,360 PATIENTS IN UNDERSERVED AREAS, 2709 01:41:18,360 --> 01:41:24,040 WHO ARE REMOTE, HOW DO WE 2710 01:41:24,040 --> 01:41:27,560 STRUCTURE RDCRN PATIENT SO -- I 2711 01:41:27,560 --> 01:41:31,320 HATE TO CALL RDCRN IN LIGHT, ARE 2712 01:41:31,320 --> 01:41:33,600 WILL PATIENTS WHERE WE PULL IN, 2713 01:41:33,600 --> 01:41:35,800 ADD DATA INTO THAT, PATIENT IN 2714 01:41:35,800 --> 01:41:37,560 DATA WHICH WE ARE SEEING MORE 2715 01:41:37,560 --> 01:41:39,080 AND MORE WITH GOOD RELIABILITY. 2716 01:41:39,080 --> 01:41:41,560 CAN WE WE USE THOSE METHODS NOT 2717 01:41:41,560 --> 01:41:47,000 ONLY TO LOWER COSTS FOR THESE 2718 01:41:47,000 --> 01:41:49,160 STUDIES CENTER FOCUSED IS YOUR 2719 01:41:49,160 --> 01:41:50,880 MOST EXPENSIVE BUT CROWD 2720 01:41:50,880 --> 01:41:52,280 SOURCING THAT TO THE PATIENT AND 2721 01:41:52,280 --> 01:41:53,640 FAMILIES, SO IT IS EASIER FOR 2722 01:41:53,640 --> 01:41:55,760 THEM TO PARTICIPATE, THERE IS 2723 01:41:55,760 --> 01:41:56,760 LESS BURDEN OF PARTICIPATION IN 2724 01:41:56,760 --> 01:41:59,320 THAT. 2725 01:41:59,320 --> 01:42:01,520 BECAUSE AS ANNE AND REBECCA BOTH 2726 01:42:01,520 --> 01:42:03,280 KNOW PARTICIPATING IN THESE 2727 01:42:03,280 --> 01:42:04,400 STUDIES IS A HIGH BURDEN. 2728 01:42:04,400 --> 01:42:06,480 AND WE SHOULD BE LOOKING AT 2729 01:42:06,480 --> 01:42:08,960 CONCRETE WAYS TO REDUCE THAT 2730 01:42:08,960 --> 01:42:10,440 BURDEN AND I THINK THAT SHOULD 2731 01:42:10,440 --> 01:42:13,560 BE ONE GOAL IN RDCRN, REDUCE 2732 01:42:13,560 --> 01:42:15,560 THAT BURDEN WHILE MAINTAINING 2733 01:42:15,560 --> 01:42:16,760 QUALITY. 2734 01:42:16,760 --> 01:42:20,200 LOVE THE RDCRN BRNS A FAN FOR A 2735 01:42:20,200 --> 01:42:21,440 LONG TIME BUT THERE'S SOME 2736 01:42:21,440 --> 01:42:24,800 THINGS WE CAN DO. 2737 01:42:24,800 --> 01:42:26,240 >> GREAT. 2738 01:42:26,240 --> 01:42:28,200 >> THANK YOU, MARSHALL. 2739 01:42:28,200 --> 01:42:30,360 >> TIINA, PLEASE. 2740 01:42:30,360 --> 01:42:33,360 >> SORRY PJ YOU GO FIRST. 2741 01:42:33,360 --> 01:42:37,040 >> ON YOUR POINT MARSHALL THE 2742 01:42:37,040 --> 01:42:39,080 SHARED MICROETIOLOGIES 2743 01:42:39,080 --> 01:42:41,360 SPECIFICALLY CALLED OUT DISEASES 2744 01:42:41,360 --> 01:42:44,320 UNDER STUDY BY RDCRN IN BOTH 2745 01:42:44,320 --> 01:42:47,720 AWARDS ESSENTIALLY INVOLVED VERY 2746 01:42:47,720 --> 01:42:51,200 CLEARLY DISEASES UNDER STUDY BY 2747 01:42:51,200 --> 01:42:55,200 RDCRN IN TIGHT CLAN RATION WITH 2748 01:42:55,200 --> 01:42:56,320 RDCRCs, I'M NOT THINKING SO 2749 01:42:56,320 --> 01:43:00,720 MUCH ABOUT THE MISSOUR MOLECULAR 2750 01:43:00,720 --> 01:43:01,720 DIAGNOSIS, LONG TERM CLINICAL 2751 01:43:01,720 --> 01:43:03,200 HISTORY DATA MANY WAYS WE CAN 2752 01:43:03,200 --> 01:43:08,560 IMPROVE ACCESS FOR THAT. 2753 01:43:08,560 --> 01:43:10,000 >> MARSHALL I WAS GOING TO SAY 2754 01:43:10,000 --> 01:43:12,360 THAT IS GOING TO COME UP IN THE 2755 01:43:12,360 --> 01:43:14,520 NEXT -- WITH THE DMCC AND WHAT 2756 01:43:14,520 --> 01:43:16,560 WE HAVE DONE IN THIS CYCLE AND 2757 01:43:16,560 --> 01:43:18,160 WHAT WE PLAN ON CONTINUING DOING 2758 01:43:18,160 --> 01:43:20,200 IN THE NEXT CYCLE. 2759 01:43:20,200 --> 01:43:24,320 >> MAYBE THAT IS A GOOD SEGUE, I 2760 01:43:24,320 --> 01:43:25,520 WANT TO MAKE SURE WE HAVE 2761 01:43:25,520 --> 01:43:27,000 COVERED THIS TOPIC ARE THERE ANY 2762 01:43:27,000 --> 01:43:32,360 OTHER DISCUSSION POINTS HERE? 2763 01:43:32,360 --> 01:43:33,840 WHAT WE ARE GOING TO DO IS NOT 2764 01:43:33,840 --> 01:43:36,840 VOTE YET, WE ARE GOING TO DO 2765 01:43:36,840 --> 01:43:38,160 NEXT ROUND HERE AND THEN VOTE 2766 01:43:38,160 --> 01:43:40,920 FOR BOTH OF THESE AT THE END. 2767 01:43:40,920 --> 01:43:43,920 IS THAT RIGHT? 2768 01:43:43,920 --> 01:43:45,240 I SEE YOUR HEAD NODDING. 2769 01:43:45,240 --> 01:43:47,120 I DON'T HEAR YOU BUT I SEE YOUR 2770 01:43:47,120 --> 01:43:47,600 HEAD NODDING. 2771 01:43:47,600 --> 01:43:50,720 IN THAT CASE -- 2772 01:43:50,720 --> 01:43:53,320 >> WE CAN DO IT THAT WAY. 2773 01:43:53,320 --> 01:43:53,920 KEEP GOING. 2774 01:43:53,920 --> 01:43:56,320 >> TIINA, DO CONCEPT NUMBER TWO, 2775 01:43:56,320 --> 01:43:59,120 THANK YOU SO MUCH. 2776 01:43:59,120 --> 01:44:01,400 >> I WANT TO THANK MARSHALL AND 2777 01:44:01,400 --> 01:44:02,200 ANMY FOR THE REMARKABLE 2778 01:44:02,200 --> 01:44:02,480 COMMENTS. 2779 01:44:02,480 --> 01:44:05,640 THANK YOU VERY MUCH. 2780 01:44:05,640 --> 01:44:06,920 NEXT COMMENT IS DATA MANAGEMENT 2781 01:44:06,920 --> 01:44:11,120 AND COORDINATING CENTER. 2782 01:44:11,120 --> 01:44:13,600 SO THE OBJECTIVE HERE IS TO 2783 01:44:13,600 --> 01:44:15,200 PROVIDE ADMINISTRATIVE CLINICAL 2784 01:44:15,200 --> 01:44:17,040 RESEARCH DATA MANAGEMENT AND 2785 01:44:17,040 --> 01:44:19,240 REGULATORY SUPPORT TO INDIVIDUAL 2786 01:44:19,240 --> 01:44:21,160 RDCRCs AND THE RDCRN. 2787 01:44:21,160 --> 01:44:24,160 WE WANT TO PROVIDE A CENTRALIZED 2788 01:44:24,160 --> 01:44:25,600 CLOUD BASED RESEARCH ENVIRONMENT 2789 01:44:25,600 --> 01:44:30,000 WITH SHARED RESOURCES FOR THE 2790 01:44:30,000 --> 01:44:30,920 RDCRN PARTICIPANTS. 2791 01:44:30,920 --> 01:44:33,120 WANT TO COORDINATE CROSS NETWORK 2792 01:44:33,120 --> 01:44:35,320 ACTIVITIES FOR THE RDCRN SUCH AS 2793 01:44:35,320 --> 01:44:37,000 WORKING GROUPS AND SPECIAL 2794 01:44:37,000 --> 01:44:38,880 INTEREST GROUPS ON A VARIETY OF 2795 01:44:38,880 --> 01:44:39,360 TOPICS. 2796 01:44:39,360 --> 01:44:41,760 THEY ARE TO SERVE AS CONDUIT OF 2797 01:44:41,760 --> 01:44:43,560 INFORMATION RELATED TO THE RARE 2798 01:44:43,560 --> 01:44:45,400 DISEASE RESEARCH BEING CONDUCTED 2799 01:44:45,400 --> 01:44:46,920 WITHIN THE NETWORK TO BOTH 2800 01:44:46,920 --> 01:44:48,040 RESEARCH COMMUNITY AND THE 2801 01:44:48,040 --> 01:44:49,440 GENERAL PUBLIC. 2802 01:44:49,440 --> 01:44:51,640 THE KEY AREAS THAT WE ARE 2803 01:44:51,640 --> 01:44:53,400 FOCUSED ON IS TO PROMOTE 2804 01:44:53,400 --> 01:44:55,520 CLINICAL TRIAL READINESS AS I 2805 01:44:55,520 --> 01:44:57,160 STATED BEFORE AND SUPPORT AND 2806 01:44:57,160 --> 01:44:58,520 FACILITATE CROSS CONSORTIA 2807 01:44:58,520 --> 01:44:59,120 COLLABORATION. 2808 01:44:59,120 --> 01:45:02,320 ONE OF OUR PRIMARY FOCUSES IS TO 2809 01:45:02,320 --> 01:45:04,520 ESTABLISH PROMOTE STANDARDS AND 2810 01:45:04,520 --> 01:45:07,480 GOOD DATA PRACTICES, EMPHASIZING 2811 01:45:07,480 --> 01:45:09,080 SCIENTIFIC RIGOR, FAIR DATA 2812 01:45:09,080 --> 01:45:10,760 PRINCIPLES AND DATA SHARING. 2813 01:45:10,760 --> 01:45:13,440 WE ALSO WANT TO DEVELOP MAINTAIN 2814 01:45:13,440 --> 01:45:15,640 A CLOUD BASED RESEARCH 2815 01:45:15,640 --> 01:45:21,480 ENVIRONMENT WITH SHARED TOOLS. 2816 01:45:21,480 --> 01:45:24,760 SO CURRENTLY THE DMCC HAS USERS 2817 01:45:24,760 --> 01:45:26,640 FROM FIVE CONTINENTS LOOKING TO 2818 01:45:26,640 --> 01:45:28,640 TAP INTO THE OTHER CONTINENTS 2819 01:45:28,640 --> 01:45:31,680 THEY PROVIDE SERVICES TO OVER 2820 01:45:31,680 --> 01:45:34,160 2000 AUTHENTICATED USERS. 2821 01:45:34,160 --> 01:45:35,280 THEY ARE BUILDING THESE TOOLS 2822 01:45:35,280 --> 01:45:38,280 FOR THE NETWORK SO RIGHT NOW 2823 01:45:38,280 --> 01:45:39,720 THERE'S 12 TOOLS FOR SHARED MET 2824 01:45:39,720 --> 01:45:41,920 WORK USE INCLUDING BIOSPECIMEN 2825 01:45:41,920 --> 01:45:46,600 TRACKING TOOLS, PEDIGREE TOOLS R 2826 01:45:46,600 --> 01:45:47,880 STUDIO AND THIS NUMBER IS 2827 01:45:47,880 --> 01:45:48,880 GROWING. 2828 01:45:48,880 --> 01:45:50,240 I'M NOT GOING TO READ THESE 2829 01:45:50,240 --> 01:45:52,200 NUMBERS, THERE'S 55 PROTOCOLS IN 2830 01:45:52,200 --> 01:45:55,160 RED CAP DATABASES, AGAIN USING 2831 01:45:55,160 --> 01:45:59,360 RED CAP THEY ARE DEVELOPING 2832 01:45:59,360 --> 01:46:00,720 STANDARDS, THERE ARE LOTS OF 2833 01:46:00,720 --> 01:46:02,720 FORMS LEGACY FORMS, VARIABLES 2834 01:46:02,720 --> 01:46:05,440 AND CURRENTLY THEY HAVE 1.5 2835 01:46:05,440 --> 01:46:07,040 TERABYTES OF DATA IN BOX. 2836 01:46:07,040 --> 01:46:09,040 AND THEY ARE ALL PUTTING THEIR 2837 01:46:09,040 --> 01:46:10,680 DATA INTO ONE ENVIRONMENT OR 2838 01:46:10,680 --> 01:46:11,640 MAJORITY OF THEM ARE PUTTING 2839 01:46:11,640 --> 01:46:13,160 DATA INTO ONE ENVIRONMENT. 2840 01:46:13,160 --> 01:46:18,960 THE RDCRN IS AGAIN ONE OF A KIND 2841 01:46:18,960 --> 01:46:19,800 TRANSLATIONAL RESEARCH NETWORK. 2842 01:46:19,800 --> 01:46:21,480 THE SUCCESS OF THE PROGRAM IS 2843 01:46:21,480 --> 01:46:22,760 ONE IN WHICH INVESTIGATORS ARE 2844 01:46:22,760 --> 01:46:24,600 WORKING IN A RESEARCH 2845 01:46:24,600 --> 01:46:25,560 ENVIRONMENT OR I LIKE TO THINK 2846 01:46:25,560 --> 01:46:27,400 OF IT AS A RESEARCH COMMUNITY, 2847 01:46:27,400 --> 01:46:30,040 ESTABLISHED BY A NETWORK USING 2848 01:46:30,040 --> 01:46:31,640 TOOLS MADE AVAILABLE. 2849 01:46:31,640 --> 01:46:33,560 SUCCESS WILL ALSO BE MEASURED BY 2850 01:46:33,560 --> 01:46:35,800 SUCCESS OF INDIVIDUAL RDCCs 2851 01:46:35,800 --> 01:46:37,960 AND USE AND ACCEPTANCE OF DMCC 2852 01:46:37,960 --> 01:46:39,200 HOSTED COLLABORATIVE TOOLS AND 2853 01:46:39,200 --> 01:46:39,760 ACTIVITIES. 2854 01:46:39,760 --> 01:46:43,800 NEXT SLIDE. 2855 01:46:43,800 --> 01:46:45,680 SO THE ROLE OF THE DMCC IS 2856 01:46:45,680 --> 01:46:48,200 SUPPORT THE RDCRN CONSORTIA AND 2857 01:46:48,200 --> 01:46:51,880 FACILITATE THEIR ABILITY TO WORK 2858 01:46:51,880 --> 01:46:52,720 FASTER, AGAIN THEY ARE 2859 01:46:52,720 --> 01:46:53,840 COORDINATING THE WORK OF 2860 01:46:53,840 --> 01:46:55,520 CONSORTIA INVESTIGATORS AND 2861 01:46:55,520 --> 01:46:58,160 PATIENT GROUPS, THEY FACILITATE 2862 01:46:58,160 --> 01:46:58,960 LONGITUDINAL STUDIES TO DEVELOP 2863 01:46:58,960 --> 01:47:00,600 A BETTER UNDERSTANDING OF RARE 2864 01:47:00,600 --> 01:47:03,800 DISEASES AND THIS IS ONE PLACE 2865 01:47:03,800 --> 01:47:06,880 WITH THE DATA STANDARDS AND IT 2866 01:47:06,880 --> 01:47:08,880 IS LIKE I SAID WHEN A STUDY 2867 01:47:08,880 --> 01:47:13,160 COMES IN, THEY START DEVELOPING 2868 01:47:13,160 --> 01:47:14,760 DATA STANDARDS DATA PRACTICES SO 2869 01:47:14,760 --> 01:47:19,760 WHEN THEY GET WHERE THEY HAVE A 2870 01:47:19,760 --> 01:47:21,720 FAIR NUMBER AND AMOUNT OF 2871 01:47:21,720 --> 01:47:22,480 LONGITUDINAL DATA THEY DON'T 2872 01:47:22,480 --> 01:47:23,360 HAVE TO REPLICATE. 2873 01:47:23,360 --> 01:47:24,960 WHEN THEY GO TO FDA THEY 2874 01:47:24,960 --> 01:47:26,440 SHOULDN'T HAVE TO GO BACK AND 2875 01:47:26,440 --> 01:47:28,760 REPLICATE THE DATA OR REDO THE 2876 01:47:28,760 --> 01:47:31,640 STUDY OR RE-EVALUATE THINGS. 2877 01:47:31,640 --> 01:47:33,640 THEY WORK IN A COST EFFECTIVE 2878 01:47:33,640 --> 01:47:36,600 MANNER BY PROVIDING SHARED TOOLS 2879 01:47:36,600 --> 01:47:38,160 AT WORK ENVIRONMENT CONSORTIA 2880 01:47:38,160 --> 01:47:39,480 USE SO INVESTIGATORS ARE USING 2881 01:47:39,480 --> 01:47:40,920 FUNDS THEY GET FROM THE GRANTS 2882 01:47:40,920 --> 01:47:44,360 NOT TO BUY LICENSES TO USE BUT 2883 01:47:44,360 --> 01:47:45,760 PUTTING TOWARDS A SCIENCE. 2884 01:47:45,760 --> 01:47:47,760 HIGH QUALITY RESEARCH ON AND 2885 01:47:47,760 --> 01:47:50,160 DATA AND REALLY EMPHASIZING 2886 01:47:50,160 --> 01:47:52,080 SCIENTIFIC RIGOR, FAIR 2887 01:47:52,080 --> 01:47:53,480 PRINCIPLES, AND BROAD REGULATORY 2888 01:47:53,480 --> 01:47:55,360 STANDARDS, I CAN'T EMPHASIZE 2889 01:47:55,360 --> 01:47:56,120 THAT ENOUGH. 2890 01:47:56,120 --> 01:47:57,160 THAT THAT'S WHAT WHICH ARE 2891 01:47:57,160 --> 01:47:58,560 FOCUSING ON. 2892 01:47:58,560 --> 01:48:01,840 AND ALSO FACILITATE DATA 2893 01:48:01,840 --> 01:48:02,400 SHARING. 2894 01:48:02,400 --> 01:48:04,720 BY PROVIDING DATA SHARING 2895 01:48:04,720 --> 01:48:06,480 ENVIRONMENTS THAT GET ACCESS AS 2896 01:48:06,480 --> 01:48:08,040 APPROPRIATE FOR THE BROADER RARE 2897 01:48:08,040 --> 01:48:09,160 DISEASE RESEARCH COMMUNITY AND 2898 01:48:09,160 --> 01:48:10,320 OTHERS. 2899 01:48:10,320 --> 01:48:13,720 AN EXCITING PART OF THE DMCC FOR 2900 01:48:13,720 --> 01:48:17,560 THIS COLLABORATION IS OUR 2901 01:48:17,560 --> 01:48:18,880 COLLABORATION WITH INFORMATION 2902 01:48:18,880 --> 01:48:20,880 TECHNOLOGY AND RESOURCES BRANCH. 2903 01:48:20,880 --> 01:48:22,320 I WOULD LIKE TO CALL OUT SAM 2904 01:48:22,320 --> 01:48:25,600 MICHAELS AND HIS TEAM FOR THE 2905 01:48:25,600 --> 01:48:26,600 GREAT WORK THEY HAVE BEEN DOING 2906 01:48:26,600 --> 01:48:27,760 WITH US TO ESTABLISH THIS. 2907 01:48:27,760 --> 01:48:30,320 AND WHAT THEY REALLY DO IS 2908 01:48:30,320 --> 01:48:32,280 DEVELOPING A TOOL -- TWO PRONG 2909 01:48:32,280 --> 01:48:35,600 CLOUD BASED WORK SPACE FOR RDCRN 2910 01:48:35,600 --> 01:48:38,040 RESEARCHERS SO FIRST AN 2911 01:48:38,040 --> 01:48:39,160 OPERATIONAL ENVIRONMENT WHERE 2912 01:48:39,160 --> 01:48:43,560 INDIVIDUAL CONSORTIUMS WORK 2913 01:48:43,560 --> 01:48:44,600 IMAGINE YOUR SPACE IN THE CLOUD 2914 01:48:44,600 --> 01:48:46,920 YOU HAVE ROOM FOR YOUR 2915 01:48:46,920 --> 01:48:47,840 CONSORTIA, DATA COLLECTED THERE 2916 01:48:47,840 --> 01:48:48,920 ALL YOUR INFORMATION HAVE, 2917 01:48:48,920 --> 01:48:50,840 THERE'S TOOLS YOU CAN USE IN 2918 01:48:50,840 --> 01:48:51,960 THIS ENVIRONMENT SHOULD YOU 2919 01:48:51,960 --> 01:48:56,560 CHOOSE TO AND IT IS THE ACTIVE 2920 01:48:56,560 --> 01:48:57,160 OPERATIONAL SITE. 2921 01:48:57,160 --> 01:48:58,080 THE SECOND ENVIRONMENT THAT WE 2922 01:48:58,080 --> 01:48:59,520 ARE IN THE PROCESS OF BUILDING 2923 01:48:59,520 --> 01:49:01,320 RIGHT NOW IS THE DATA SHARING 2924 01:49:01,320 --> 01:49:01,880 ENVIRONMENT. 2925 01:49:01,880 --> 01:49:03,680 THIS IS WHAT MANY OF YOU BROUGHT 2926 01:49:03,680 --> 01:49:05,200 UP FOR THE DATA WILL BE SHARED 2927 01:49:05,200 --> 01:49:08,080 WITH A BROADER COMMUNITY, IN A 2928 01:49:08,080 --> 01:49:09,160 CLOUD ENVIRONMENT WITH TOOLS FOR 2929 01:49:09,160 --> 01:49:11,160 QUERYING THE DATA AND DATA 2930 01:49:11,160 --> 01:49:13,240 ANALYSIS PROVIDED AND THAT 2931 01:49:13,240 --> 01:49:14,360 ENVIRONMENT IS GOVERNED BY 2932 01:49:14,360 --> 01:49:15,920 NCATS. 2933 01:49:15,920 --> 01:49:19,960 SO WE ARE REALLY BUILDING THIS 2934 01:49:19,960 --> 01:49:21,920 SHARING ENVIRONMENT THAT IS 2935 01:49:21,920 --> 01:49:24,720 GOVERNED BY NCATS BUT IS MANAGED 2936 01:49:24,720 --> 01:49:26,400 BY THE DATA COORDINATING CENTER 2937 01:49:26,400 --> 01:49:27,840 AND WHOEVER HAS THAT GRANT. 2938 01:49:27,840 --> 01:49:33,240 NEXT SLIDE PLEASE. 2939 01:49:33,240 --> 01:49:34,800 SO GENERAL QUESTIONS, ARE WILL 2940 01:49:34,800 --> 01:49:37,120 ANY OTHER AREAS OF STUDY OR 2941 01:49:37,120 --> 01:49:40,200 EMPHASIS OR SUPPORT BUT THE DMCC 2942 01:49:40,200 --> 01:49:41,920 SHOULD CONSIDER THAT WOULD HELP 2943 01:49:41,920 --> 01:49:43,040 ADVANCE RARE DISEASE RESEARCH. 2944 01:49:43,040 --> 01:49:46,400 AND ONE THING SOMEHOW GOT LEFT 2945 01:49:46,400 --> 01:49:47,720 OFF THE SLIDES IS THAT WE ARE 2946 01:49:47,720 --> 01:49:52,400 ALSO WORKING WITH THE GROUPS 2947 01:49:52,400 --> 01:49:55,400 CYCLING OFF SO THEIR DATA WILL 2948 01:49:55,400 --> 01:49:57,200 BE IN THE CLOUD ENVIRONMENT BUT 2949 01:49:57,200 --> 01:49:59,560 WILL STILL HAVE ACCESS FOR A 2950 01:49:59,560 --> 01:50:00,840 PERIOD OF TIME TO GO UP THERE 2951 01:50:00,840 --> 01:50:02,080 AND USE THAT ENVIRONMENT, HAVE 2952 01:50:02,080 --> 01:50:06,280 ACCESS FOR THEIR DATA AND WORK 2953 01:50:06,280 --> 01:50:10,880 IN THAT SO IT IS OFTEN USE THE 2954 01:50:10,880 --> 01:50:12,200 ANALOGY WE ARE GRADUATING AND 2955 01:50:12,200 --> 01:50:14,640 GOING OFF TO COLLEGE BUT THEY 2956 01:50:14,640 --> 01:50:16,760 CAN STILL COME BACK AND DO 2957 01:50:16,760 --> 01:50:19,040 LAUNDRY AT HOME, THEY ARE 2958 01:50:19,040 --> 01:50:21,440 BECOMING MORE INDEPENDENT BUT 2959 01:50:21,440 --> 01:50:23,000 NOT TOTALLY CUTTING THEM LOOSE 2960 01:50:23,000 --> 01:50:23,960 AND THERE WILL BE SUPPORT FOR 2961 01:50:23,960 --> 01:50:26,560 THEM. 2962 01:50:26,560 --> 01:50:28,560 I OPEN THIS UP FOR COMMENTS. 2963 01:50:28,560 --> 01:50:29,520 JONI. 2964 01:50:29,520 --> 01:50:31,120 >> THANK YOU, TIINA. 2965 01:50:31,120 --> 01:50:34,000 WE HAVE BECKY ANNIE AND MATTHIAS 2966 01:50:34,000 --> 01:50:37,520 SO BECKY YOU ARE UP FIRST AGAIN. 2967 01:50:37,520 --> 01:50:39,960 >> THANK YOU FOR THAT OVERVIEW. 2968 01:50:39,960 --> 01:50:43,160 SO IN A SLIDE BEFORE THIS, YOU 2969 01:50:43,160 --> 01:50:45,720 SAY DATA ACCESSIBILITY FOR THE 2970 01:50:45,720 --> 01:50:47,480 RESEARCH COMMUNITY, I WOULD 2971 01:50:47,480 --> 01:50:49,720 SLASH THE WORD RESEARCH AND 2972 01:50:49,720 --> 01:50:52,920 RUELY IS THE BROAD COMMUNITY OF 2973 01:50:52,920 --> 01:50:53,880 STAKEHOLDERS. 2974 01:50:53,880 --> 01:50:55,520 THAT INCLUDES SCIENTISTS, 2975 01:50:55,520 --> 01:50:58,520 INCLUDES PAGES, PATIENTS, AND 2976 01:50:58,520 --> 01:51:02,040 INCLUDES POLICY MAKERS, BROADLY. 2977 01:51:02,040 --> 01:51:04,720 I THINK THAT'S CRITICAL AND 2978 01:51:04,720 --> 01:51:06,200 NEEDS TO UNDERLINE THIS. 2979 01:51:06,200 --> 01:51:07,760 THE SECOND THING I DIDN'T SEE 2980 01:51:07,760 --> 01:51:10,280 BROUGHT OUT EITHER IN READING 2981 01:51:10,280 --> 01:51:12,080 THAT CONCEPT PROPOSAL, OR 2982 01:51:12,080 --> 01:51:13,720 LOOKING AT THE SLIDES OR IN YOUR 2983 01:51:13,720 --> 01:51:16,240 PRESENTATION I REALIZE THESE GET 2984 01:51:16,240 --> 01:51:18,360 TRUNCATED TO KEEP THEM WITHIN 2985 01:51:18,360 --> 01:51:22,920 THE TIME BUT IT IS BRINGING IN 2986 01:51:22,920 --> 01:51:24,840 AND ADDITIONAL DATA SETS THAT 2987 01:51:24,840 --> 01:51:29,040 ALLOW US TO UTILIZE NATIONAL 2988 01:51:29,040 --> 01:51:32,120 DATA TO UNDERSTAND THE 2989 01:51:32,120 --> 01:51:33,880 COMMUNITIES AND NATURAL HISTORY 2990 01:51:33,880 --> 01:51:39,800 AS WELL, I THINK ONE OF THE 2991 01:51:39,800 --> 01:51:41,760 ISSUES THAT I WILL GRAPPLE WITH 2992 01:51:41,760 --> 01:51:43,920 ARE MARGINALIZED POPULATIONS, 2993 01:51:43,920 --> 01:51:45,120 INDIVIDUALINGS WHO DON'T HAVE 2994 01:51:45,120 --> 01:51:50,160 THE SAME LEVEL OF CONTACT WHO 2995 01:51:50,160 --> 01:51:53,800 CARE FOR RARE DISEASE. 2996 01:51:53,800 --> 01:51:57,120 SO TO UNDERSTAND AND MAKE SURE 2997 01:51:57,120 --> 01:51:59,320 THAT THE DATA WE ARE PRESENTING 2998 01:51:59,320 --> 01:52:02,160 IS PUT WITHIN CONTEXT SOCIAL 2999 01:52:02,160 --> 01:52:06,600 DETERMINANTS OF HEALTH, AND 3000 01:52:06,600 --> 01:52:08,800 OTHER THINGS THAT IMPACT EQUITY, 3001 01:52:08,800 --> 01:52:11,480 I THINK REALLY IMMEDIATE TO MAKE 3002 01:52:11,480 --> 01:52:13,560 A RESPONSIBILITY OF THE DATA 3003 01:52:13,560 --> 01:52:16,600 COORDINATING CENTER NOT JUST FOR 3004 01:52:16,600 --> 01:52:18,160 THE INDIVIDUAL SPECIFIC NETWORKS 3005 01:52:18,160 --> 01:52:20,080 BUT FOR DATA COORDINATING CENTER 3006 01:52:20,080 --> 01:52:21,560 BRING MANY THOSE KIND OF 3007 01:52:21,560 --> 01:52:22,560 NATIONAL DATA. 3008 01:52:22,560 --> 01:52:24,960 AND BE ABLE NOW TO START TO LINK 3009 01:52:24,960 --> 01:52:26,560 THOSE TOGETHER. 3010 01:52:26,560 --> 01:52:30,360 WHICH ALLOWS I THINK MUCH MORE 3011 01:52:30,360 --> 01:52:32,160 UNBIASED DATA TO MOVE FORWARD TO 3012 01:52:32,160 --> 01:52:34,960 HELP MAKE SOME OF OUR DECISIONS. 3013 01:52:34,960 --> 01:52:37,960 AND THEN THE LAST THING IS 3014 01:52:37,960 --> 01:52:42,520 DIGITAL DIVIDE, OR DIGITAL 3015 01:52:42,520 --> 01:52:43,960 EQUITY, AND NOT JUST DATA 3016 01:52:43,960 --> 01:52:46,000 EQUITY. 3017 01:52:46,000 --> 01:52:47,680 BECAUSE IN MAKING THINGS 3018 01:52:47,680 --> 01:52:48,920 ACCESSIBLE WE HAVE TO RECOGNIZE 3019 01:52:48,920 --> 01:52:51,560 THAT THERE IS A WHOLE SET OF 3020 01:52:51,560 --> 01:52:52,240 ISSUES. 3021 01:52:52,240 --> 01:52:54,840 THAT INCLUDES MAKING THE 3022 01:52:54,840 --> 01:52:57,600 INFORMATION USABLE AND 3023 01:52:57,600 --> 01:52:58,880 UNDERSTANDABLE BY OTHERS. 3024 01:52:58,880 --> 01:53:02,960 SO I WOULD ENCOURAGE AS THAT 3025 01:53:02,960 --> 01:53:06,240 DATA SHARING IS EXPANDED THAT WE 3026 01:53:06,240 --> 01:53:09,400 THINK ABOUT HOW TO DO THAT IN A 3027 01:53:09,400 --> 01:53:12,760 WAY THAT MEANINGFUL INFORMATION 3028 01:53:12,760 --> 01:53:16,120 CAN BE OBTAINED RATHER THIS THAN 3029 01:53:16,120 --> 01:53:18,400 JUST LARGE SETS OF NUMBERS WHICH 3030 01:53:18,400 --> 01:53:20,600 CAN OFTEN BE MISINTERPRETED AND 3031 01:53:20,600 --> 01:53:23,760 LEAD TO ERRONEOUS ASSUMPTIONS 3032 01:53:23,760 --> 01:53:26,520 GOING FORWARD. 3033 01:53:26,520 --> 01:53:29,800 I THINK THAT THERE'S LOTS OF 3034 01:53:29,800 --> 01:53:32,040 THINGS EXCITING ABOUT THIS AND 3035 01:53:32,040 --> 01:53:33,560 HEARING YOU TALK WHEN WE BROUGHT 3036 01:53:33,560 --> 01:53:34,760 IT UP BEFORE WE SHOULD HAVE 3037 01:53:34,760 --> 01:53:37,240 BROUGHT IT UP ABOUT SOME OF THE 3038 01:53:37,240 --> 01:53:41,480 INTERACTIONS WORKING WITH 3039 01:53:41,480 --> 01:53:43,480 COMMUNITY PATIENT ADVOCACY 3040 01:53:43,480 --> 01:53:44,520 GROUPS AND OTHERS. 3041 01:53:44,520 --> 01:53:49,320 I WOULD ENCOURAGE THAT STAYS AN 3042 01:53:49,320 --> 01:53:50,480 IMPORTANT INTERNAL AD VEUZRY 3043 01:53:50,480 --> 01:53:52,440 BOARD AND SOME OF THAT TRAINING 3044 01:53:52,440 --> 01:53:55,080 OF HOW TO USE DATA AND HOW TO 3045 01:53:55,080 --> 01:53:56,560 ADDRESS SOME OF THESE ISSUES 3046 01:53:56,560 --> 01:53:57,960 THAT ARE GIVEN AS TOOLS FOR 3047 01:53:57,960 --> 01:54:00,360 OTHER PEOPLE TO BE ABLE TO 3048 01:54:00,360 --> 01:54:03,240 DISSEMINATE TO THE POPULATIONS 3049 01:54:03,240 --> 01:54:05,360 THEY WORK WITH. 3050 01:54:05,360 --> 01:54:07,280 >> THANK YOU BECKY. 3051 01:54:07,280 --> 01:54:09,960 THAT WAS HELPFUL. 3052 01:54:09,960 --> 01:54:11,240 GO AHEAD TIINA. 3053 01:54:11,240 --> 01:54:13,240 >> THAT IS GREAT BUT ONE THING I 3054 01:54:13,240 --> 01:54:16,200 WASN'T ABLE TO PUT INTO THE 3055 01:54:16,200 --> 01:54:18,840 PRESENTATION IS AS WE ARE 3056 01:54:18,840 --> 01:54:19,920 DEVELOPING THESE TOOLS IN THE 3057 01:54:19,920 --> 01:54:21,760 DATA STANDARDS WHAT ARE THE MCC 3058 01:54:21,760 --> 01:54:24,320 HAS BEEN DOING IS REACHING OUT 3059 01:54:24,320 --> 01:54:25,840 TO OTHER GROUPS SO WHATEVER THEY 3060 01:54:25,840 --> 01:54:28,800 ARE BUILDING, WE ARE TRYING OUR 3061 01:54:28,800 --> 01:54:32,120 BEST TO MAKE SURE THAT IT IS 3062 01:54:32,120 --> 01:54:33,680 INTEROPERABLE, IF OTHER 3063 01:54:33,680 --> 01:54:34,720 ORGANIZATIONS ARE COLLECTING A 3064 01:54:34,720 --> 01:54:39,000 LOT OF RARE DISEASE RESEARCH, WE 3065 01:54:39,000 --> 01:54:40,760 WANT THE BRING THOSE DATA SETS 3066 01:54:40,760 --> 01:54:41,160 TOGETHER. 3067 01:54:41,160 --> 01:54:42,760 SO THERE ARE -- THEY HAVE BEEN 3068 01:54:42,760 --> 01:54:44,840 HAVING CONVERSATIONS WITH 3069 01:54:44,840 --> 01:54:46,960 EXTERNAL GROUPS, HARD TO PUT ALL 3070 01:54:46,960 --> 01:54:48,880 THIS INTO THE PRESENTATION. 3071 01:54:48,880 --> 01:54:50,760 THAT WAS A VERY IMPORTANT POINT 3072 01:54:50,760 --> 01:54:52,360 AND THAT'S VERY IMPORTANT TO US 3073 01:54:52,360 --> 01:54:56,480 TO MAKE SURE THAT AND I LOVE 3074 01:54:56,480 --> 01:55:01,440 YOUR POINT ABOUT I HI -- CPAC 3075 01:55:01,440 --> 01:55:02,920 USE EVERYTHING AS WELL. 3076 01:55:02,920 --> 01:55:07,720 >> ANNIE. 3077 01:55:07,720 --> 01:55:10,120 >> TIINA, YOU JUST AGAIN TO 3078 01:55:10,120 --> 01:55:14,520 ADDRESS THE FIRST THING WANTED 3079 01:55:14,520 --> 01:55:16,160 TO BRING OUT, MAYBE YOU HAVE A 3080 01:55:16,160 --> 01:55:17,040 MAGIC SOLUTION TO THIS. 3081 01:55:17,040 --> 01:55:18,360 BUT I JUST THINK IN THE RARE 3082 01:55:18,360 --> 01:55:22,080 DISEASE COMMUNITY, AND SOME WAYS 3083 01:55:22,080 --> 01:55:23,360 WE ARE DROWBING IN DATA AND 3084 01:55:23,360 --> 01:55:25,440 OTHER WAYS WE ARE DESPERATE NEED 3085 01:55:25,440 --> 01:55:28,360 OF DATA. 3086 01:55:28,360 --> 01:55:29,960 SO MANY DATA COLLECTION EFFORTS 3087 01:55:29,960 --> 01:55:33,000 UNDERWAY AND PLACES WHERE OUR 3088 01:55:33,000 --> 01:55:34,360 DATA IS BEING STORED AND 3089 01:55:34,360 --> 01:55:36,160 CHECKED: AND WHAT WE 3090 01:55:36,160 --> 01:55:37,560 DESPERATELY NEED IS FOR DATA 3091 01:55:37,560 --> 01:55:40,720 COLLECTION EFFORTS TO COME 3092 01:55:40,720 --> 01:55:43,360 TOGETHER AND HAVE LONGEVITY AS 3093 01:55:43,360 --> 01:55:44,560 MARSHALL WAS SAYING AND US TO 3094 01:55:44,560 --> 01:55:48,040 KNOW THAT THAT DATA IS GOING TO 3095 01:55:48,040 --> 01:55:50,720 BE STORED AND CONTINUED. 3096 01:55:50,720 --> 01:55:53,240 SO TO THE DEGREE YOU ARE ABLE, I 3097 01:55:53,240 --> 01:55:55,760 REALLY URGE THIS DATA COLLECTION 3098 01:55:55,760 --> 01:56:01,720 EFFORTS YOU LOOK AT THE MAJOR 3099 01:56:01,720 --> 01:56:04,360 COLLABORATIVE EFFORT CPA AND 3100 01:56:04,360 --> 01:56:06,320 NORD HAS DATA PLATFORMS ALL OF 3101 01:56:06,320 --> 01:56:07,960 YOUR PAGs PROBABLY HAVE THEIR 3102 01:56:07,960 --> 01:56:09,400 OWN REGISTRIES AND MANY OF O 3103 01:56:09,400 --> 01:56:10,480 THOSE EFFORTS ARE ALSO LOOKING 3104 01:56:10,480 --> 01:56:13,080 AT HOW TO PULL IN INDIVIDUAL 3105 01:56:13,080 --> 01:56:16,720 PATIENT EHR INTO BACK END DATA 3106 01:56:16,720 --> 01:56:20,160 POINTS THEY ARE COLLECTING. 3107 01:56:20,160 --> 01:56:21,760 THAT IS CRITICAL SO WE DON'T 3108 01:56:21,760 --> 01:56:22,880 HAVE INDIVIDUAL PATIENTS 3109 01:56:22,880 --> 01:56:24,040 FAMILIES AT THE END OF THE DAY 3110 01:56:24,040 --> 01:56:27,960 AFTER A LONG DAY OF LIVING OUR 3111 01:56:27,960 --> 01:56:31,520 LIVES WHICH ARE HARD RIGHT NOW 3112 01:56:31,520 --> 01:56:32,520 ANYWAY, PEOPLE ARE ENTERING 3113 01:56:32,520 --> 01:56:35,000 THEIR DATA INTO THE (INAUDIBLE) 3114 01:56:35,000 --> 01:56:36,240 DATA OR THINKING ABOUT WHERE 3115 01:56:36,240 --> 01:56:38,680 DATA IS COLLECTED OR WHERE 3116 01:56:38,680 --> 01:56:40,480 INFORMATION HAS TO BE UPLOADED. 3117 01:56:40,480 --> 01:56:43,360 SO JUST TO BE RESPECTFUL OF THAT 3118 01:56:43,360 --> 01:56:45,240 TIME AND ENERGY AND MAKE SURE 3119 01:56:45,240 --> 01:56:47,440 EVERYONE IS DOING ALL THAT. 3120 01:56:47,440 --> 01:56:49,160 THEN JUST MY OTHER THING I KNOW 3121 01:56:49,160 --> 01:56:51,160 THIS IS SORT OF FEEL LIKE IT IS 3122 01:56:51,160 --> 01:56:53,160 BECOMING MY RESOUNDING THEME BUT 3123 01:56:53,160 --> 01:56:54,480 ONE OF THE THINGS YOU HAVE 3124 01:56:54,480 --> 01:56:58,200 TALKED ABOUT I THINK YOU SAID IS 3125 01:56:58,200 --> 01:56:59,560 ONE OF THE GOALS IS WE WOULDN'T 3126 01:56:59,560 --> 01:57:00,920 HAVE TO RECOLLECT THE DATA WHEN 3127 01:57:00,920 --> 01:57:02,480 WE GET TO THE FDA. 3128 01:57:02,480 --> 01:57:05,680 WE DO IT RIGHT THE FIRST TIME, I 3129 01:57:05,680 --> 01:57:06,960 AM IN COMPLETE AGREEMENT WITH 3130 01:57:06,960 --> 01:57:09,520 BUT I URGE US TOT TO NOT THINK 3131 01:57:09,520 --> 01:57:11,360 OF THE FDA REGULATORY REVIEW AS 3132 01:57:11,360 --> 01:57:13,440 OUR END POINT AND END GOAL FOR 3133 01:57:13,440 --> 01:57:16,200 PATIENT COMMUNITIES, IF WHICH 3134 01:57:16,200 --> 01:57:18,160 CAN'T ACCESS IT IN PROVIDER 3135 01:57:18,160 --> 01:57:25,560 CAN'T CONSIDER IT, THEN NONE OF 3136 01:57:25,560 --> 01:57:26,840 THIS IS WORTH IT AT THE END OF 3137 01:57:26,840 --> 01:57:28,120 THE DAY, SO THE END GOAL NEEDS 3138 01:57:28,120 --> 01:57:29,440 TORR WHETHER OR NOT WE HAVE 3139 01:57:29,440 --> 01:57:30,760 CONSIDERED WHETHER OR NOT THIS 3140 01:57:30,760 --> 01:57:32,800 WILL INFORM DECISION MAKING IN 3141 01:57:32,800 --> 01:57:33,960 THE PAYER ENVIRONMENT SO WE 3142 01:57:33,960 --> 01:57:36,680 DON'T HAVE TO RECOLLECT 3143 01:57:36,680 --> 01:57:38,560 DIFFERENT DATA FOR CMS OR 3144 01:57:38,560 --> 01:57:40,560 MEDICAID STATES OR WHOM EVER IS 3145 01:57:40,560 --> 01:57:42,720 GOING TO BE ADDING A SEPARATE, 3146 01:57:42,720 --> 01:57:43,960 WE ARE NOW IN THIS SPACE WHERE 3147 01:57:43,960 --> 01:57:47,400 WE ARE ALMOST IN THE U.S. TO 3148 01:57:47,400 --> 01:57:48,840 EUROPEAN MODEL TO ADJUDICATION 3149 01:57:48,840 --> 01:57:49,720 SYSTEMS, WE DON'T HAVE THAT 3150 01:57:49,720 --> 01:57:51,440 HERE. 3151 01:57:51,440 --> 01:57:53,120 SO I THINK NCATS IS WELL POISED 3152 01:57:53,120 --> 01:57:56,760 TO BE THINKING ABOUT THESE 3153 01:57:56,760 --> 01:57:58,240 ISSUES AND BRINGING STAKEHOLDERS 3154 01:57:58,240 --> 01:58:01,800 IN TO THESE PROCESSES. 3155 01:58:01,800 --> 01:58:05,000 >> THANK YOU, ANNIE. 3156 01:58:05,000 --> 01:58:06,040 COULDN'T AGREE MORE. 3157 01:58:06,040 --> 01:58:07,840 MATTHIAS YOU ARE THIRD HERE. 3158 01:58:07,840 --> 01:58:09,000 LET YOU HAVE AN OPTION. 3159 01:58:09,000 --> 01:58:12,360 >> HUE SO MUCH. 3160 01:58:12,360 --> 01:58:14,040 FRIDAY AFTERNOON 3 P.M. AFTER 3161 01:58:14,040 --> 01:58:15,920 LONG WEEK LONG DAY BUT THIS IS 3162 01:58:15,920 --> 01:58:17,720 MOST IMPORTANT DISCUSSION FROM 3163 01:58:17,720 --> 01:58:19,280 MY VANTAGE POINT WE HAVE HAD 3164 01:58:19,280 --> 01:58:20,760 OVER THE LAST COUPLE OF DAYS. 3165 01:58:20,760 --> 01:58:24,360 THIS IS REALLY RARE DISEASE 3166 01:58:24,360 --> 01:58:26,040 SPACE, WE ARE AT A UNIQUE 3167 01:58:26,040 --> 01:58:28,240 OPPORTUNITY IN TIME AND IT HAS 3168 01:58:28,240 --> 01:58:29,080 NEARLY TRANSCENDED THE 3169 01:58:29,080 --> 01:58:31,560 DISCUSSION OVER THE LAST DAY AS 3170 01:58:31,560 --> 01:58:35,160 WELL, REDEFINING WHAT THE HUMANS 3171 01:58:35,160 --> 01:58:37,760 ARE ON MONOCLONAL CELL BASED 3172 01:58:37,760 --> 01:58:39,960 LEVEL OPENS OPPORTUNITIES TO 3173 01:58:39,960 --> 01:58:42,600 TRANSCEND THAT KNOWLEDGE TO 3174 01:58:42,600 --> 01:58:44,960 THERAPIES AND PJ FOR PRESENTING 3175 01:58:44,960 --> 01:58:46,400 IF WE HAVE TOOLS IN HAND TO 3176 01:58:46,400 --> 01:58:48,840 EFFECTIVELY TARGET TO COVER OR 3177 01:58:48,840 --> 01:58:50,600 ELIMINATE FUNCTION OF MOLECULES 3178 01:58:50,600 --> 01:58:53,360 AND CELLS CAUSING OUR DISEASE 3179 01:58:53,360 --> 01:58:54,720 (INAUDIBLE) IF WE ALLOW 3180 01:58:54,720 --> 01:58:56,920 KNOWLEDGE STATES. 3181 01:58:56,920 --> 01:58:58,480 THAT IS WHAT CAN AND NEEDS TO DO 3182 01:58:58,480 --> 01:59:02,480 AND WHAT THE DNCC WILL BE HAVE 3183 01:59:02,480 --> 01:59:04,760 TO DO THAT. 3184 01:59:04,760 --> 01:59:08,040 AND IT IS VERY EXCITING, WITH 3185 01:59:08,040 --> 01:59:11,800 TIINA, FOR THOSE THAT ARE RARE 3186 01:59:11,800 --> 01:59:13,960 -- STUDY NETWORK ONE OF THE 3187 01:59:13,960 --> 01:59:15,160 RDCRCs WHICH WILL BE 3188 01:59:15,160 --> 01:59:16,680 SUNSETTING SO I DON'T HAVE ANY 3189 01:59:16,680 --> 01:59:20,680 CONFLICT OF INTEREST. 3190 01:59:20,680 --> 01:59:22,240 REALLY STARTING TO SEE A CULTURE 3191 01:59:22,240 --> 01:59:24,680 CHANGE IN THE RDCRC AS WELL 3192 01:59:24,680 --> 01:59:27,400 INITIALLY A LOT OF THE NETWORKS 3193 01:59:27,400 --> 01:59:29,640 CAME ON EXPERTS WHO DEDICATED 3194 01:59:29,640 --> 01:59:34,840 THEIR LIVES TO A CERTAIN DISEASE 3195 01:59:34,840 --> 01:59:36,160 AND (INAUDIBLE) WHERE THE 3196 01:59:36,160 --> 01:59:39,240 DISEASE WAS GOING IN THE 3197 01:59:39,240 --> 01:59:39,880 TREATMENT CHANNEL. 3198 01:59:39,880 --> 01:59:42,360 WE ARE SEEING THE TRANSDEN SENSE 3199 01:59:42,360 --> 01:59:45,080 NOW TO DATA RICH FIELDS WHERE 3200 01:59:45,080 --> 01:59:48,680 DATA GAINS VALUE IF SHARED 3201 01:59:48,680 --> 01:59:50,880 WIDELY AND THAT DATA ISOLATION 3202 01:59:50,880 --> 01:59:52,800 NEEDS TO BE VIGOROUSLY OPENED UP 3203 01:59:52,800 --> 01:59:54,880 THAT IS A HARD -- THAT IS A 3204 01:59:54,880 --> 01:59:56,200 SENSITIVE PROCESS FOR MANY, MANY 3205 01:59:56,200 --> 01:59:58,960 REASONS. 3206 01:59:58,960 --> 02:00:02,240 TIINA HAS TO BE CONGRATULATED 3207 02:00:02,240 --> 02:00:04,920 WITH HER SYSTEMS IMPERSONATION 3208 02:00:04,920 --> 02:00:07,520 WITH THE CURRENT RDCRN TO GET TO 3209 02:00:07,520 --> 02:00:14,360 THAT TRIAL SO THE NEXT RFA TO 3210 02:00:14,360 --> 02:00:17,680 HAVE CULTURE CHANGE TAKE PLACE. 3211 02:00:17,680 --> 02:00:19,200 CLEARLY INCENTIVIZING ACTIVITIES 3212 02:00:19,200 --> 02:00:22,200 BETWEEN THE DIFFERENT RDCRCs 3213 02:00:22,200 --> 02:00:24,760 AND FOR THAT I SEE ANOTHER 3214 02:00:24,760 --> 02:00:26,760 NETWORK WHERE LEADERSHIP WAS 3215 02:00:26,760 --> 02:00:29,400 OPPORTUNITY POOLS, MEANING 3216 02:00:29,400 --> 02:00:33,000 DEDICATED RESOURCES FOR SPECIFIC 3217 02:00:33,000 --> 02:00:34,200 INTERACTIVITIES INSIDE THE NET 3218 02:00:34,200 --> 02:00:37,640 WORK WHO ARE NOT PREDEFINED BUT 3219 02:00:37,640 --> 02:00:39,320 AVAILABLE AND DEPLOYABLE IN A 3220 02:00:39,320 --> 02:00:41,200 GIVEN FUNDING SITE. 3221 02:00:41,200 --> 02:00:44,520 CAN BE VERY HELPFUL, IT HAS BEEN 3222 02:00:44,520 --> 02:00:46,600 FOR THE MEDICINE PROJECT WHERE 3223 02:00:46,600 --> 02:00:48,920 LEADS DATA COORDINATING CENTER, 3224 02:00:48,920 --> 02:00:50,920 HAS BEEN HELPFUL FOR US TO 3225 02:00:50,920 --> 02:00:53,320 REALLY BE NIMBLE AND FLEXIBLE TO 3226 02:00:53,320 --> 02:00:55,840 THE LATEST DEVELOPMENT AND IF 3227 02:00:55,840 --> 02:00:58,400 THIS IS CONSISTENT WITH THE 3228 02:00:58,400 --> 02:01:01,240 CONCEPT FOR YOUR RFA HAVE 3229 02:01:01,240 --> 02:01:01,960 (INAUDIBLE) SHOULD BE 3230 02:01:01,960 --> 02:01:04,040 IMPLEMENTED. 3231 02:01:04,040 --> 02:01:05,640 WF HAD DISCUSSION ON STANDARDS 3232 02:01:05,640 --> 02:01:08,560 AN ONTOLOGIES ESTABLISHING 3233 02:01:08,560 --> 02:01:10,320 EXPANDING YOUR KNOWLEDGE AFTER 3234 02:01:10,320 --> 02:01:11,360 WHAT YOU ALREADY HAVE MANY PLACE 3235 02:01:11,360 --> 02:01:13,320 IN N CATS WHERE YOU BRING 3236 02:01:13,320 --> 02:01:14,360 KNOWLEDGE TOGETHER. 3237 02:01:14,360 --> 02:01:17,960 WITH THE INTERNATIONAL 3238 02:01:17,960 --> 02:01:19,120 COMMUNITY, THE FOUNDRY IS A 3239 02:01:19,120 --> 02:01:20,680 GREAT PLACE TO START HUMAN 3240 02:01:20,680 --> 02:01:22,200 PHENOTYPE ONTOLOGY WHICH IS 3241 02:01:22,200 --> 02:01:25,760 INVOLVED WITH IRD IRC IS A GREAT 3242 02:01:25,760 --> 02:01:28,760 PLACE TO TRAND SEND NOW THE 3243 02:01:28,760 --> 02:01:30,600 BASIC SCIENCE CELL BIOLOGY 3244 02:01:30,600 --> 02:01:31,960 MONOCLONAL BIOLOGY UP TO THE 3245 02:01:31,960 --> 02:01:36,280 DISEASE PHENOTYPING ERTS. 3246 02:01:36,280 --> 02:01:37,480 PHENOTYPING EFFORTS. 3247 02:01:37,480 --> 02:01:38,520 ANOTHER INTERESTING FEATURE, HOW 3248 02:01:38,520 --> 02:01:42,640 CAN YOU GET THE DATA IF -- 3249 02:01:42,640 --> 02:01:43,240 READY. 3250 02:01:43,240 --> 02:01:44,960 YES AND NO, MY CONCERN IS YOU 3251 02:01:44,960 --> 02:01:48,560 HAVE THESE DISCUSSIONS ALSO WITH 3252 02:01:48,560 --> 02:01:50,240 THE RETURN WITHOUT 3253 02:01:50,240 --> 02:01:51,760 DISCUSSIONINGS WITH A LOT OF OUR 3254 02:01:51,760 --> 02:01:56,920 WORK IN OUR NETWORKS. 3255 02:01:56,920 --> 02:01:58,320 WE ARE CAREFULLY CONSIDERING YES 3256 02:01:58,320 --> 02:02:02,400 YOU WOULD LIKE TO BRING THE DATA 3257 02:02:02,400 --> 02:02:03,720 BACK TO STUDY PARTICIPANTS. 3258 02:02:03,720 --> 02:02:06,960 BUT BE CAREFUL AND OBVIOUSLY 3259 02:02:06,960 --> 02:02:09,040 INSTITUTE OF MEDICINE SPENT TIME 3260 02:02:09,040 --> 02:02:09,960 AND EFFORT THINKING HOW TO DO 3261 02:02:09,960 --> 02:02:13,760 THAT. 3262 02:02:13,760 --> 02:02:16,360 IF BEESTABLISH QUALITY STANDARD 3263 02:02:16,360 --> 02:02:19,080 REQUIRES OUR RESEARCH STREAMS 3264 02:02:19,080 --> 02:02:20,480 TOP CLEAR EFFORTS AND OUR 3265 02:02:20,480 --> 02:02:23,240 EFFORTS VERY QUICKLY WILL 3266 02:02:23,240 --> 02:02:25,600 DIMINISH IN THEIR -- BUT FINDING 3267 02:02:25,600 --> 02:02:27,720 THE RIGHT BALANCE IN ACQUIRING 3268 02:02:27,720 --> 02:02:30,480 DATA WITH STANDARDS, WITH 3269 02:02:30,480 --> 02:02:33,000 TOOLBOXES HIGHEST QUALITY 3270 02:02:33,000 --> 02:02:34,560 POSSIBLE WITHOUT SUFFOCATING 3271 02:02:34,560 --> 02:02:38,080 RESEARCH EFFORTS, IS A VERY FINE 3272 02:02:38,080 --> 02:02:40,680 BALANCE. 3273 02:02:40,680 --> 02:02:42,560 STAKEHOLDERS IN PARTICULAR, TO 3274 02:02:42,560 --> 02:02:46,760 PEOPLE LIKE MYSELF INVESTIGATORS 3275 02:02:46,760 --> 02:02:48,880 AND BRANCHES WHO HAVE TO KEEP 6 3276 02:02:48,880 --> 02:02:49,760 SIDES OPERATIONAL AND MOVE 3277 02:02:49,760 --> 02:02:52,760 FORWARD AND KEEP DATA COMING IN, 3278 02:02:52,760 --> 02:02:54,840 WILL BE RELEVANT IN THAT 3279 02:02:54,840 --> 02:02:56,760 CONTEXT. 3280 02:02:56,760 --> 02:03:02,560 SO I WOULD REALLY ASK YOU TO 3281 02:03:02,560 --> 02:03:03,960 CONTINUE TO PUSH FOR CULTURE 3282 02:03:03,960 --> 02:03:05,720 CHANGE BUT BE INNOVATIVE TOWARDS 3283 02:03:05,720 --> 02:03:07,680 THE BASIC SCIENCE PERSPECTIVE, 3284 02:03:07,680 --> 02:03:09,680 THIS IS CHEERILY A CLINICAL 3285 02:03:09,680 --> 02:03:12,320 TRANSLATIONAL NETWORK. 3286 02:03:12,320 --> 02:03:13,360 BUT THE OPPORTUNITIES COMING 3287 02:03:13,360 --> 02:03:16,160 FROM THE BASIC SCIENCE ARE 3288 02:03:16,160 --> 02:03:18,040 TRANSFORMATIVE AND DMCC SHOULD 3289 02:03:18,040 --> 02:03:22,680 BE ABLE TO LIFT AND TRANSCEND 3290 02:03:22,680 --> 02:03:26,400 THESE ASPECTS AS WELL. 3291 02:03:26,400 --> 02:03:28,360 >> THANK YOU FOR THAT MATTHIAS. 3292 02:03:28,360 --> 02:03:29,800 COULDN'T AGREE MORE. 3293 02:03:29,800 --> 02:03:32,920 IT IS A CHANGING ENVIRONMENT AND 3294 02:03:32,920 --> 02:03:34,560 HA INCLUDES THE CULTURE ITSELF 3295 02:03:34,560 --> 02:03:36,160 IN THIS TYPE OF DATA WE ARE 3296 02:03:36,160 --> 02:03:36,680 WORKING WITH. 3297 02:03:36,680 --> 02:03:38,880 THAT'S SO IMPORTANT THAT WE KEEP 3298 02:03:38,880 --> 02:03:41,320 POLLING ON THOSE AS WELL TO KEEP 3299 02:03:41,320 --> 02:03:43,320 ADVANCING THEM AND DOING SO 3300 02:03:43,320 --> 02:03:44,760 RESPONSIBLY AND APPROPRIATELY. 3301 02:03:44,760 --> 02:03:48,200 TIINA, DID YOU HAVE SOMETHING? 3302 02:03:48,200 --> 02:03:50,320 >> I WAS NODDING MY HEAD IN 3303 02:03:50,320 --> 02:03:54,120 AGREEMENT AND EVALUATING EVERY 3304 02:03:54,120 --> 02:03:56,520 WORD COMING OUT -- THANK YOU SO 3305 02:03:56,520 --> 02:03:57,040 MUCH. 3306 02:03:57,040 --> 02:04:00,240 >> THANK YOU FOR THE LINKS AS 3307 02:04:00,240 --> 02:04:01,840 WELL. 3308 02:04:01,840 --> 02:04:04,360 FROM OUR PERSPECTIVE AT NCATS 3309 02:04:04,360 --> 02:04:05,760 ONE OF THE GREAT THINGS ABOUT 3310 02:04:05,760 --> 02:04:07,760 THIS PROGRAM TOO IS THE 3311 02:04:07,760 --> 02:04:09,160 IMPORTANCE OF THE COLLABORATIONS 3312 02:04:09,160 --> 02:04:11,080 WE HAVE ACROSS THE NIH AND SO I 3313 02:04:11,080 --> 02:04:13,680 SEE JILL MORRIS' HAND UP, FROM 3314 02:04:13,680 --> 02:04:17,400 HIND AND WONDERFUL TO -- FOR 3315 02:04:17,400 --> 02:04:18,880 EXAMPLE, AN INCREDIBLE PARTNER, 3316 02:04:18,880 --> 02:04:23,160 SO PLEASE GO AHEAD AND -- 3317 02:04:23,160 --> 02:04:29,480 >> I AM A PROGRAM DIRECTOR AT 3318 02:04:29,480 --> 02:04:33,200 NINDS FOR THE RDCRN PROGRAM, TO 3319 02:04:33,200 --> 02:04:34,560 NCATS. 3320 02:04:34,560 --> 02:04:37,880 SO WE FUND NUMEROUS RDCRCs AND 3321 02:04:37,880 --> 02:04:47,840 CO-FUND DMCC, I APPRECIATE 3322 02:04:47,840 --> 02:04:48,960 BRIETDING THEM DOWN. 3323 02:04:48,960 --> 02:04:51,280 ONE THING TO EMPHASIZE THAT'S 3324 02:04:51,280 --> 02:04:56,120 NEW SINCE RDCRN IS TIINA PUT 3325 02:04:56,120 --> 02:04:57,360 TOGETHER FOR DATA STANDARD 3326 02:04:57,360 --> 02:05:01,760 WORKING GROUPS, AND SO THEY ARE 3327 02:05:01,760 --> 02:05:03,600 FOR THE DIFFERENT TYPES OF DATA 3328 02:05:03,600 --> 02:05:04,800 STANDARDS, THEY MEET ON A 3329 02:05:04,800 --> 02:05:06,360 MONTHLY BASIS, AND THEN THE 3330 02:05:06,360 --> 02:05:08,560 WHOLE GROUP, THE WHOLE DATA 3331 02:05:08,560 --> 02:05:09,960 STANDARD GROUP MEETS ON 3332 02:05:09,960 --> 02:05:11,240 QUARTERLY BASIS. 3333 02:05:11,240 --> 02:05:16,240 I SAT IN ON ONE LAST WEEK. 3334 02:05:16,240 --> 02:05:18,280 AND THEY WERE -- IT IS A SCOM BY 3335 02:05:18,280 --> 02:05:19,960 NATION OF -- COMBINATION OF 3336 02:05:19,960 --> 02:05:21,040 INVESTIGATORS AN PATIENT 3337 02:05:21,040 --> 02:05:22,720 ADVOCACY GROUPS AND DMCC ON 3338 02:05:22,720 --> 02:05:23,160 THESE. 3339 02:05:23,160 --> 02:05:28,200 AND THEY WERE TALKING ABOUT 3340 02:05:28,200 --> 02:05:30,920 DIFFERENT SKILLS IN THE 3341 02:05:30,920 --> 02:05:32,000 TELEHEALTH TYPE METHOD. 3342 02:05:32,000 --> 02:05:34,240 SO THEY ARE WORKING AS A TEAM TO 3343 02:05:34,240 --> 02:05:35,800 ESTABLISH DATA STANDARDS THAT I 3344 02:05:35,800 --> 02:05:39,440 THINK WILL BE USEFUL PUSHING 3345 02:05:39,440 --> 02:05:40,760 THESE STUDIES FORWARD TO CLINIC. 3346 02:05:40,760 --> 02:05:42,000 SO I JUST WANTED TO EMPHASIZE 3347 02:05:42,000 --> 02:05:44,480 THAT. 3348 02:05:44,480 --> 02:05:46,160 >> THANK YOU FOR THAT. 3349 02:05:46,160 --> 02:05:51,200 THANK YOU FOR YOUR PARTICIPATION 3350 02:05:51,200 --> 02:05:51,960 RDCRN TOO. 3351 02:05:51,960 --> 02:05:53,520 GREAT TO HAVE WONDERFUL PARTNERS 3352 02:05:53,520 --> 02:05:54,520 ACROSS THE NIH ON THIS. 3353 02:05:54,520 --> 02:05:56,120 SO IMPORTANT. 3354 02:05:56,120 --> 02:05:59,200 >> HAPPY TO DO IT. 3355 02:05:59,200 --> 02:06:01,200 I SEE MARSHALL YOUR HAND UP AND 3356 02:06:01,200 --> 02:06:02,520 GIVE YOU THE LAST WORD FOR 3357 02:06:02,520 --> 02:06:04,520 DISCUSSION AND I THINK WE NEED 3358 02:06:04,520 --> 02:06:07,080 TO MOVE ON. 3359 02:06:07,080 --> 02:06:10,240 >> TWO QUICK THINGS ONE FOR 3360 02:06:10,240 --> 02:06:11,680 REGULAR DATA COORDINATION, IS 3361 02:06:11,680 --> 02:06:13,120 THERE SOMEONE FROM AGENCY LIKE 3362 02:06:13,120 --> 02:06:15,200 FDA ALSO THERE SAYING HEY, THIS 3363 02:06:15,200 --> 02:06:17,960 IS NICE BUT THIS DOESN'T WORK. 3364 02:06:17,960 --> 02:06:20,760 TIINA, DO Y'ALL HAVE -- I 3365 02:06:20,760 --> 02:06:22,520 FIGURED Y'ALL HAD SOMEBODY IN 3366 02:06:22,520 --> 02:06:22,720 THERE. 3367 02:06:22,720 --> 02:06:26,960 >> WE REACH OUT BROADLY TO 3368 02:06:26,960 --> 02:06:29,720 VARIETY OF GROUPS COLLECTING ANY 3369 02:06:29,720 --> 02:06:32,240 KIND OF DATA, WE TALK TO THE FDA 3370 02:06:32,240 --> 02:06:33,800 BUT LIKE MATTHIAS SAID THEY ARE 3371 02:06:33,800 --> 02:06:36,160 NOT THE ONLY GAME IN TOWN. 3372 02:06:36,160 --> 02:06:39,440 WE ARE TRYING -- SO WE ARE 3373 02:06:39,440 --> 02:06:41,360 TRYING TO BALANCE. 3374 02:06:41,360 --> 02:06:44,080 WE ARE EQUAL OPPORTUNITY REACHER 3375 02:06:44,080 --> 02:06:46,160 OUTERS. 3376 02:06:46,160 --> 02:06:47,680 WE TALK TO EVERYONE DIFFERENT 3377 02:06:47,680 --> 02:06:48,880 ORGANIZATIONS THAT ARE BRINGING 3378 02:06:48,880 --> 02:06:49,480 THEIR DATA TOGETHER. 3379 02:06:49,480 --> 02:06:51,440 WE DON'T WANT TO REPLICATE. 3380 02:06:51,440 --> 02:06:53,200 WE WANT THINGS TO BE 3381 02:06:53,200 --> 02:06:54,160 INTEROPERABLE AND THAT IS THE 3382 02:06:54,160 --> 02:06:57,760 KEY WORD THAT WE USE. 3383 02:06:57,760 --> 02:07:00,360 >> CURIOUS TOO, THAT 3384 02:07:00,360 --> 02:07:01,600 INTEROPERABILITY IN EVERY -- 3385 02:07:01,600 --> 02:07:03,200 >> UNDERSTOOD BUT AT THE END OF 3386 02:07:03,200 --> 02:07:04,920 THE DAY IT WOULD BE NICE WHEN WE 3387 02:07:04,920 --> 02:07:06,160 DO THESE CLINICAL TRIALS THAT 3388 02:07:06,160 --> 02:07:08,240 MOVE FORWARD THAT THE DATA CAN 3389 02:07:08,240 --> 02:07:10,120 IF NOTHING ELSE BE USED TO 3390 02:07:10,120 --> 02:07:11,760 ESTABLISH NATURAL HISTORY. 3391 02:07:11,760 --> 02:07:13,120 SO CONTROL THE STUDY OR 3392 02:07:13,120 --> 02:07:15,080 SOMETHING LIKE THAT. 3393 02:07:15,080 --> 02:07:17,800 >> THE FDA MARSHALL, I HAVE TO 3394 02:07:17,800 --> 02:07:20,760 SAY, IS THEY HAVE BEEN VERY 3395 02:07:20,760 --> 02:07:22,200 ACTIVELY PARTICIPATING IN OUR 3396 02:07:22,200 --> 02:07:24,560 MEETINGS COMING TO SESSIONS, I 3397 02:07:24,560 --> 02:07:26,160 HAVE A MONTHLY INDIVIDUAL 3398 02:07:26,160 --> 02:07:29,040 MEETING WITH WHERE WE TALK ABOUT 3399 02:07:29,040 --> 02:07:32,480 RDCRN AND WHAT WE CAN DO. 3400 02:07:32,480 --> 02:07:37,240 >> I HELPED TRAIN PETRELLA. 3401 02:07:37,240 --> 02:07:39,200 LAST IS A PHILOSOPHICAL THING. 3402 02:07:39,200 --> 02:07:40,360 INDULGE ME A SECOND HERE. 3403 02:07:40,360 --> 02:07:41,560 Y'ALL HAVE CREATED SOMETHING 3404 02:07:41,560 --> 02:07:44,000 THAT DOESN'T FIT THE CLASSIC NIH 3405 02:07:44,000 --> 02:07:47,040 MODEL OF FIVE YEAR PROJECTS AND 3406 02:07:47,040 --> 02:07:50,120 THINGS LIKE THAT, WHAT Y'ALL 3407 02:07:50,120 --> 02:07:51,200 CREATEDDED IS A COLLECTION OF 3408 02:07:51,200 --> 02:07:53,520 DATA THAT IS ALMOST A NATIONAL 3409 02:07:53,520 --> 02:07:56,080 TREASURE. 3410 02:07:56,080 --> 02:07:58,720 AND AS THESE PROGRAMS SUN DOWN 3411 02:07:58,720 --> 02:08:00,840 AND THOUGH IT SOUNDS LIKE THE 3412 02:08:00,840 --> 02:08:02,400 PLANS YOU CAN ACCESS FOR A 3413 02:08:02,400 --> 02:08:05,040 WHILE, I REALLY LIKEIAL TO COME 3414 02:08:05,040 --> 02:08:07,000 BACK -- Y'ALL TO COME BACK AND 3415 02:08:07,000 --> 02:08:08,960 THINK THROUGH HOW DO MAKE SURE 3416 02:08:08,960 --> 02:08:12,160 THIS STAYS EXTANT FOR A LONG 3417 02:08:12,160 --> 02:08:12,560 PERIOD OF TIME. 3418 02:08:12,560 --> 02:08:14,360 THE AMOUNT OF TREASURER AND 3419 02:08:14,360 --> 02:08:16,360 EFFORT FROM BOTH PATIENT 3420 02:08:16,360 --> 02:08:17,680 COMMUNITY, AS WELL AS THE 3421 02:08:17,680 --> 02:08:18,920 RESEARCH COMMUNITY TO CREATE 3422 02:08:18,920 --> 02:08:20,280 THESE DATA SETS IS MASSIVE. 3423 02:08:20,280 --> 02:08:22,280 THIS IS SOMETHING THAT I WOULD 3424 02:08:22,280 --> 02:08:25,440 LOVE TO SEE AROUND FOR VERY LONG 3425 02:08:25,440 --> 02:08:25,800 TIME. 3426 02:08:25,800 --> 02:08:27,040 SO MANY QUESTIONS WE HAVE COME 3427 02:08:27,040 --> 02:08:29,040 UP WITH 15 YEARS FROM NOW, 20 3428 02:08:29,040 --> 02:08:30,760 YEARS FROM NOW THAT MAY EXIST IN 3429 02:08:30,760 --> 02:08:33,240 THESE DATA SETS THAT ARE CHECKED 3430 02:08:33,240 --> 02:08:35,360 I DON'T EXPECT AN ANSWER BUT I 3431 02:08:35,360 --> 02:08:36,440 WANT YOU TO THINK ABOUT THIS 3432 02:08:36,440 --> 02:08:37,680 DIFFERENTLY THAN YOU DO YOUR 3433 02:08:37,680 --> 02:08:39,880 CLASSIC FIVE YEARS OR HOWEVER 3434 02:08:39,880 --> 02:08:41,600 LONG PROJECT IS GOING TO LAST. 3435 02:08:41,600 --> 02:08:43,840 >> I THOUGHT IN MY HEAD WE HAVE 3436 02:08:43,840 --> 02:08:45,240 CENTERS OF EXCELLENCE HERE AT 3437 02:08:45,240 --> 02:08:46,400 THE NIH, MAYBE WE NEED A GNASH 3438 02:08:46,400 --> 02:08:48,600 THAT WILL TREASURE DESIGNATION 3439 02:08:48,600 --> 02:08:50,560 AS WELL IN SOME WAY. 3440 02:08:50,560 --> 02:08:54,640 SO IT IS A GREAT IDEA, GREAT 3441 02:08:54,640 --> 02:08:55,800 POINT SO THANK YOU. 3442 02:08:55,800 --> 02:08:57,960 I WILL THINK ABOUT THAT MORE. 3443 02:08:57,960 --> 02:09:00,960 >> JODY, IF SAM MICHAELS IS ON, 3444 02:09:00,960 --> 02:09:03,280 IF THIS SOMETHING WE CAN TALK 3445 02:09:03,280 --> 02:09:04,560 ABOUT QUITE A LOT, I DON'T KNOW 3446 02:09:04,560 --> 02:09:08,280 IF SAM IS AVAILABLE TO SAY THIS 3447 02:09:08,280 --> 02:09:10,720 IS HOW WE ARE THINKING OF 3448 02:09:10,720 --> 02:09:12,760 BUILDING AND SUPPORTING THINGS. 3449 02:09:12,760 --> 02:09:15,480 >> THANK YOU. 3450 02:09:15,480 --> 02:09:16,280 >> THANKS. 3451 02:09:16,280 --> 02:09:17,960 >> WITH THAT I THINK WE WILL GO 3452 02:09:17,960 --> 02:09:19,760 AHEAD AND MOVE ON TO THE VOTE 3453 02:09:19,760 --> 02:09:20,160 HERE. 3454 02:09:20,160 --> 02:09:22,000 SO ANNA, I WILL TURN IT OVER TO 3455 02:09:22,000 --> 02:09:26,680 YOU TO DO THE NEXT STEP. 3456 02:09:26,680 --> 02:09:27,920 >> THIS IS JONI. 3457 02:09:27,920 --> 02:09:30,360 SO WE WILL VOTE ON TWO CONCEPTS 3458 02:09:30,360 --> 02:09:31,560 FROM OFFICE OF RARE DISEASE 3459 02:09:31,560 --> 02:09:32,600 RESEARCH. 3460 02:09:32,600 --> 02:09:36,280 THE FIRST ONE IS FOR THE RDCRN 3461 02:09:36,280 --> 02:09:37,680 CONSORTIA, MAY I HAVE A MOTION. 3462 02:09:37,680 --> 02:09:39,520 >> SO MOVED. 3463 02:09:39,520 --> 02:09:40,040 >> SECOND. 3464 02:09:40,040 --> 02:09:41,240 >> SECOND. 3465 02:09:41,240 --> 02:09:43,480 >> ALL IN FAVOR. 3466 02:09:43,480 --> 02:09:43,720 >> AYE. 3467 02:09:43,720 --> 02:09:43,960 >> AYE. 3468 02:09:43,960 --> 02:09:45,760 >> AYE. 3469 02:09:45,760 --> 02:09:49,640 >> ANY OPPOSED? 3470 02:09:49,640 --> 02:09:52,560 ANY ABSTENTIONS? 3471 02:09:52,560 --> 02:09:53,800 THANK YOU. 3472 02:09:53,800 --> 02:09:56,560 THE CONCEPT FOR THE RDCRN CON 3473 02:09:56,560 --> 02:09:58,120 SOR HA IS APPROVED. 3474 02:09:58,120 --> 02:09:59,440 NEXT MAY I HAVE A MOTION TO 3475 02:09:59,440 --> 02:10:02,440 ADVANCE THE DMCC CONCEPT FOR THE 3476 02:10:02,440 --> 02:10:04,560 RARE DISEASE CLINICAL RESEARCH 3477 02:10:04,560 --> 02:10:05,080 NETWORK? 3478 02:10:05,080 --> 02:10:06,200 >> MOVE. 3479 02:10:06,200 --> 02:10:07,600 SECOND. 3480 02:10:07,600 --> 02:10:08,120 SECOND. 3481 02:10:08,120 --> 02:10:09,280 >> ALL IN FAVOR. 3482 02:10:09,280 --> 02:10:10,240 >> AYE. 3483 02:10:10,240 --> 02:10:12,680 >> AYE. 3484 02:10:12,680 --> 02:10:15,680 >> ANY OPPOSED? 3485 02:10:15,680 --> 02:10:18,560 ANY ABSTENTIONS? 3486 02:10:18,560 --> 02:10:22,160 THANK YOU VERY MUCH, ORDR RDCRN 3487 02:10:22,160 --> 02:10:24,080 CONCEPTS HAVE BEEN APPROVED. 3488 02:10:24,080 --> 02:10:26,200 >> THANK YOU, APPRECIATE THAT 3489 02:10:26,200 --> 02:10:28,880 AND THANK YOU FOR THE VOTES. 3490 02:10:28,880 --> 02:10:30,880 WE ARE ROUNDING THIRD HERE ON 3491 02:10:30,880 --> 02:10:32,600 OUR WAY HOME FOR THE REST OF THE 3492 02:10:32,600 --> 02:10:33,800 AFTERNOON AND WE HAVE A COUPLE 3493 02:10:33,800 --> 02:10:36,320 MORE THINGS WE NEED YOUR HELP 3494 02:10:36,320 --> 02:10:36,880 ON. 3495 02:10:36,880 --> 02:10:39,560 THE FIRST IS THE PRESENTATION 3496 02:10:39,560 --> 02:10:42,200 CALLED THE NCATS TRIENNIAL 3497 02:10:42,200 --> 02:10:43,360 INCLUSION REPORT AS MENTIONED 3498 02:10:43,360 --> 02:10:45,160 YESTERDAY, THIS IS ROUTINE BUT 3499 02:10:45,160 --> 02:10:47,800 NOT ROUTINE, DONE EVERY THREE 3500 02:10:47,800 --> 02:10:48,800 YEARS. 3501 02:10:48,800 --> 02:10:50,800 DR. VALERY GORDON IS OUR ACTING 3502 02:10:50,800 --> 02:10:52,160 DIRECTOR OF OFFICE OF 3503 02:10:52,160 --> 02:10:53,600 TRANSLATIONAL MEDICINE AND SHE'S 3504 02:10:53,600 --> 02:10:55,280 ALSO SENIOR ADVISOR FOR CLINICAL 3505 02:10:55,280 --> 02:10:56,720 RESEARCH AND INCLUSION POLICY 3506 02:10:56,720 --> 02:10:58,920 OFFICER HERE AT N CATS SO SHE IS 3507 02:10:58,920 --> 02:11:01,120 GOING TO DO THIS PRESENTATION 3508 02:11:01,120 --> 02:11:03,000 AND HOPE TO GIVE YOUR -- GET 3509 02:11:03,000 --> 02:11:04,920 YOUR INPUT ON THAT. 3510 02:11:04,920 --> 02:11:09,360 VALERY, PLEASE GO AHEAD. 3511 02:11:09,360 --> 02:11:11,680 >> WE CAN HEAR YOU. 3512 02:11:11,680 --> 02:11:16,120 >> I'M HERE TO PRESENT THE NCATS 3513 02:11:16,120 --> 02:11:18,760 TRIENNIAL REPORT ON MONITORING 3514 02:11:18,760 --> 02:11:20,280 ADHERENCE TO NIH POLICIES ON 3515 02:11:20,280 --> 02:11:23,880 INCLUSION OF WOMEN MINORITIES. 3516 02:11:23,880 --> 02:11:24,520 IN CLINICAL RESEARCH. 3517 02:11:24,520 --> 02:11:25,840 COULD I HAVE THE NEXT SLIDE 3518 02:11:25,840 --> 02:11:28,160 PLEASE. 3519 02:11:28,160 --> 02:11:30,040 IN CASE YOU ARE WONDERING WHY 3520 02:11:30,040 --> 02:11:32,160 I'M PRESENTING THIS. 3521 02:11:32,160 --> 02:11:34,800 THE PUBLIC HEALTH SERVICE ACT 3522 02:11:34,800 --> 02:11:36,560 REQUIRES NIH TO ENSURE INCLUSION 3523 02:11:36,560 --> 02:11:39,320 OF WOMEN AND MINORITIES IN NIH 3524 02:11:39,320 --> 02:11:42,840 FUNDED RESEARCH. 3525 02:11:42,840 --> 02:11:44,360 IN THE MANNER APPROPRIATE TO 3526 02:11:44,360 --> 02:11:45,120 SCIENTIFIC QUESTIONS UNDER 3527 02:11:45,120 --> 02:11:45,520 STUDY. 3528 02:11:45,520 --> 02:11:46,800 THAT IS A REALLY IMPORTANT 3529 02:11:46,800 --> 02:11:50,120 POINT. 3530 02:11:50,120 --> 02:11:52,120 NIH THEN REPORTS AGGREGATE DATA 3531 02:11:52,120 --> 02:11:55,080 TO CONGRESS IN A TRIENNIAL NIH 3532 02:11:55,080 --> 02:11:57,600 REPORT SO NCATS DATA WILL BE 3533 02:11:57,600 --> 02:12:01,760 COMBINED WITH ALL OTHER IC DATA. 3534 02:12:01,760 --> 02:12:02,800 FINAL REPORT. 3535 02:12:02,800 --> 02:12:05,120 BUT AS PART OF THE REPORT IN 3536 02:12:05,120 --> 02:12:08,560 PROCESS, EACH NIH IC NEEDS TO 3537 02:12:08,560 --> 02:12:10,160 PRESENT THEIR DATA TO THEIR 3538 02:12:10,160 --> 02:12:14,240 ADVISORY COUNCIL. 3539 02:12:14,240 --> 02:12:16,320 AND MY -- THE DATA TABLES I'M 3540 02:12:16,320 --> 02:12:20,200 GOING TO SHOW ARE COMPLICATED. 3541 02:12:20,200 --> 02:12:23,680 AND MY RESPONSIBILITY TO FOCUS 3542 02:12:23,680 --> 02:12:26,320 ATTENTION ON THE MAJOR POINTS IN 3543 02:12:26,320 --> 02:12:28,200 EACH TABLE RATHER THAN GOING 3544 02:12:28,200 --> 02:12:29,960 OVER ALL THE DETAILS WHICH WOULD 3545 02:12:29,960 --> 02:12:33,400 TAKE A REALLY LONG TIME. 3546 02:12:33,400 --> 02:12:34,680 SO THE INFORMATION THESE TABLES 3547 02:12:34,680 --> 02:12:36,680 ARE IN YOUR COUNCIL NOTE BOOKS 3548 02:12:36,680 --> 02:12:39,640 SO IF YOU CAN ENCOURAGE THEM TO 3549 02:12:39,640 --> 02:12:41,440 READ THEM AT YOUR LEISURE. 3550 02:12:41,440 --> 02:12:43,000 NCATS TAKES DATA SERIOUSLY AND 3551 02:12:43,000 --> 02:12:45,360 WORK TO ENSURE WE ARE INCLUDING 3552 02:12:45,360 --> 02:12:47,080 RELEVANT POPULATIONS. 3553 02:12:47,080 --> 02:12:49,560 IN A STUDY WE SUPPORT. 3554 02:12:49,560 --> 02:12:53,560 NO ACTION BY COUNCIL IS 3555 02:12:53,560 --> 02:12:55,120 REQUIRED, SIMPLY REQUIRED TO 3556 02:12:55,120 --> 02:12:56,680 PRESENT DATA TO YOU. 3557 02:12:56,680 --> 02:13:02,280 NEXT SLIDE PLEASE. 3558 02:13:02,280 --> 02:13:06,240 SO I'M HERE TO REVIEW THE FISCAL 3559 02:13:06,240 --> 02:13:08,480 YEAR 2019 TO 2021 ENROLLMENT 3560 02:13:08,480 --> 02:13:10,840 DATA AND OFFICE OF NIH OFFICE OF 3561 02:13:10,840 --> 02:13:13,920 EXTRAMURAL RESEARCH, REQUIRES 3562 02:13:13,920 --> 02:13:15,440 CERTIFICATION BY IC DIRECTORS 3563 02:13:15,440 --> 02:13:17,560 THAT THE DATA ARE ACCEPT NL FOR 3564 02:13:17,560 --> 02:13:18,560 INCLUSION -- ACCEPTABLE FOR 3565 02:13:18,560 --> 02:13:19,920 INCLUSION MANY THIS TRIENNIAL 3566 02:13:19,920 --> 02:13:21,440 REPORT. 3567 02:13:21,440 --> 02:13:24,320 I'M GOING TO NOTE THAT THE DATA 3568 02:13:24,320 --> 02:13:26,600 TABLES REFLECT THE INFORMATION 3569 02:13:26,600 --> 02:13:28,000 COLLECTED AS OF THE DATE THE 3570 02:13:28,000 --> 02:13:33,440 REPORT WAS RUN AND THAT WAS IN 3571 02:13:33,440 --> 02:13:34,960 MAY AND NOVEMBER SO CHANGES ARE 3572 02:13:34,960 --> 02:13:37,640 MADE BY AWARDEES OR NCATS FOR 3573 02:13:37,640 --> 02:13:39,040 ONGOING STUDIES, THESE DATA 3574 02:13:39,040 --> 02:13:44,360 CHANGE ALL TIME. 3575 02:13:44,360 --> 02:13:46,360 ALTHOUGH FISCAL YEAR 2021 DATA 3576 02:13:46,360 --> 02:13:48,000 ARE LOCKED FOR CHANGES. 3577 02:13:48,000 --> 02:13:53,280 NEXT SLIDE PLEASE. 3578 02:13:53,280 --> 02:13:55,000 MAJORITY OF NCATS SUPPORTED 3579 02:13:55,000 --> 02:13:56,560 CLINICAL RESEARCH, MAJORITY 3580 02:13:56,560 --> 02:13:59,080 TAKES THE FORM OF FORMAL 3581 02:13:59,080 --> 02:14:00,880 PARTNERSHIPS WITH OTHER NIH 3582 02:14:00,880 --> 02:14:02,360 ICs YOU HEARD ABOUT SOME OF 3583 02:14:02,360 --> 02:14:05,680 THEM TODAY. 3584 02:14:05,680 --> 02:14:08,880 TO AVOID DUPLICATION OF -- 3585 02:14:08,880 --> 02:14:10,960 PARTNERING I SEE NOT NCATS 3586 02:14:10,960 --> 02:14:12,560 REPORTS INCLUSION ENROLLMENT 3587 02:14:12,560 --> 02:14:12,920 DATA. 3588 02:14:12,920 --> 02:14:17,640 THIS MEANS THAT NCATS NUMBERS 3589 02:14:17,640 --> 02:14:19,320 ARE MODEST WHEN COMPARED TO 3590 02:14:19,320 --> 02:14:21,760 OTHER NIH ICs AND CREATES SOME 3591 02:14:21,760 --> 02:14:26,160 VARIABILITY IN NUMBERS BUT I 3592 02:14:26,160 --> 02:14:28,000 WANT TO SHOW EXAMPLES OF NCATS 3593 02:14:28,000 --> 02:14:32,600 PROGRAMS FOR WHICH OTHER NIH 3594 02:14:32,600 --> 02:14:35,360 ICs REPORT ENROLLMENT DATA 3595 02:14:35,360 --> 02:14:40,320 INCLUDE COMMUNITY ENGAGEMENT 3596 02:14:40,320 --> 02:14:43,960 ALLIANCE AGAINST COVID-19 3597 02:14:43,960 --> 02:14:45,520 DISPARITIES, HEEL INITIATIVES TO 3598 02:14:45,520 --> 02:14:47,560 END ADDICTION LONG TERM, NCATS 3599 02:14:47,560 --> 02:14:50,120 TRIAL INNOVATION NETWORK AND 3600 02:14:50,120 --> 02:14:52,040 RDCRN OUTLET YOU HEARD GREAT 3601 02:14:52,040 --> 02:14:53,280 DEAL TODAY. 3602 02:14:53,280 --> 02:14:55,200 EXAMPLES OF NCATS PROGRAMS FOR 3603 02:14:55,200 --> 02:14:58,920 WHICH NCATS ENROLLMENT DATA ARE 3604 02:14:58,920 --> 02:15:00,160 -- SO THOSE ARE THE NUMBERS YOU 3605 02:15:00,160 --> 02:15:02,520 SEE ARE THE CTSA PROGRAM 3606 02:15:02,520 --> 02:15:05,960 COLLABORATIVE INNOVATION AWARDS 3607 02:15:05,960 --> 02:15:07,200 SMALL BUSINESS INNOVATION 3608 02:15:07,200 --> 02:15:12,440 RESEARCH AWARDS SBIRs, AND 3609 02:15:12,440 --> 02:15:13,960 CTSA PILOT STUDIES I COULDN'T 3610 02:15:13,960 --> 02:15:16,440 GET EXACT NUMBER BUT OVER 330 3611 02:15:16,440 --> 02:15:19,960 FOR FISCAL YEAR 2021. 3612 02:15:19,960 --> 02:15:21,760 THEY ARE SMALL, I NEED TO NOTE 3613 02:15:21,760 --> 02:15:23,640 THAT THE NCATS INTRAMURAL 3614 02:15:23,640 --> 02:15:25,080 PROGRAM DIDN'T SUPPORT ANY 3615 02:15:25,080 --> 02:15:26,240 CLINICAL RESEARCH DURING THIS 3616 02:15:26,240 --> 02:15:27,880 PERIOD. 3617 02:15:27,880 --> 02:15:29,880 BUT THEY ARE PLANNING CLINICAL 3618 02:15:29,880 --> 02:15:32,360 TRIALS STEMMING FROM DTR 3619 02:15:32,360 --> 02:15:33,280 RESEARCH IN THIS CURRENT FISCAL 3620 02:15:33,280 --> 02:15:35,520 YEAR. 3621 02:15:35,520 --> 02:15:39,080 NEXT SLIDE PLEASE. 3622 02:15:39,080 --> 02:15:41,360 SO OVER THE PAST THREE YEARS, 3623 02:15:41,360 --> 02:15:42,760 I'M GOING TO GET TO THE DATA IN 3624 02:15:42,760 --> 02:15:43,680 A MINUTE. 3625 02:15:43,680 --> 02:15:46,520 NCATS HAS APPLIED INNOVATIVE 3626 02:15:46,520 --> 02:15:47,840 TRANSLATIONAL SCIENCE PARADIGM 3627 02:15:47,840 --> 02:15:50,280 TO ADDRESS HEALTH DISPARITIES, 3628 02:15:50,280 --> 02:15:52,360 IT UNDERSERVE AND VULNERABLE 3629 02:15:52,360 --> 02:15:54,520 POPULATIONS. 3630 02:15:54,520 --> 02:15:57,160 SPECIFICALLY WE LEVERAGE CTSA TO 3631 02:15:57,160 --> 02:15:59,640 PARTICIPATE IN NIH WIDE EFFORTS 3632 02:15:59,640 --> 02:16:01,560 TO EXPLORE WAYS TO INCREASE 3633 02:16:01,560 --> 02:16:06,320 INCLUSION OF THESE POPULATIONS. 3634 02:16:06,320 --> 02:16:08,840 AND EXAMPLES OF THESEN COLLUDE 3635 02:16:08,840 --> 02:16:12,080 THE RADX PROGRAMS, AND SEAL 3636 02:16:12,080 --> 02:16:14,680 WHICH I SPOKE ABOUT PREVIOUSLY. 3637 02:16:14,680 --> 02:16:17,120 AND ADDITIONALLY PUBLISHED A 3638 02:16:17,120 --> 02:16:19,440 NOTICE TO PROMOTE EXPANSION OF 3639 02:16:19,440 --> 02:16:20,960 CTS EFFORTS TO ACCELERATE 3640 02:16:20,960 --> 02:16:23,080 CLINICAL TRANSLATIONAL RESEARCH 3641 02:16:23,080 --> 02:16:25,160 TO ADDRESS HEALTH DISPARITIES, 3642 02:16:25,160 --> 02:16:27,960 AND SIGNIFICANT BURDEN OF 3643 02:16:27,960 --> 02:16:29,640 CONDITIONS, DISPROPORTIONATELY 3644 02:16:29,640 --> 02:16:31,960 AFFECT RURAL MINORITY AND OTHER 3645 02:16:31,960 --> 02:16:33,160 UNDERSERVED POPULATIONS. 3646 02:16:33,160 --> 02:16:37,360 AND IN 2021 NCATS ENHANCED CTSA 3647 02:16:37,360 --> 02:16:40,800 PROGRAM GOALS TO CREATE PROVIDE 3648 02:16:40,800 --> 02:16:42,480 DISSEMINATE INNOVATIVE RESEARCH 3649 02:16:42,480 --> 02:16:45,240 PROGRAMS PARTNERSHIPS ACROSS 3650 02:16:45,240 --> 02:16:48,320 INSTITUTIONS BETWEEN THESE TO 3651 02:16:48,320 --> 02:16:49,160 ADDRESS HEALTH DISPARITIES AND 3652 02:16:49,160 --> 02:16:50,240 DELIVER BENEFITS OF 3653 02:16:50,240 --> 02:16:51,760 TRANSLATIONAL SCIENCE TO ALL SO 3654 02:16:51,760 --> 02:16:53,560 AS YOU CAN TELL WE DO TAKE THIS 3655 02:16:53,560 --> 02:16:57,360 SERIOUSLY. 3656 02:16:57,360 --> 02:17:03,960 SO DATA NEXT SLIDE PLEASE. 3657 02:17:03,960 --> 02:17:05,480 FIRST TOTAL NUMBER OF NCATS 3658 02:17:05,480 --> 02:17:06,800 AWARDS WITH RESEARCH INVOLVING 3659 02:17:06,800 --> 02:17:08,720 HUMAN SUBJECTS OVER THE THREE 3660 02:17:08,720 --> 02:17:10,040 YEAR PERIOD. 3661 02:17:10,040 --> 02:17:13,480 AND IF YOU -- AS YOU LOOK, YOU 3662 02:17:13,480 --> 02:17:19,520 WILL SEE I WON'T SHOW TAKE HOME 3663 02:17:19,520 --> 02:17:20,840 MESSAGE AT THE BOTTOM OF EACH 3664 02:17:20,840 --> 02:17:22,200 TABLE BUT NUMBER OF AWARDS IS 3665 02:17:22,200 --> 02:17:25,160 LOWER MANY FISCAL YEAR 2021, 3666 02:17:25,160 --> 02:17:29,040 WHEN COMPARED TO PREVIOUS TWO 3667 02:17:29,040 --> 02:17:31,000 YEARS DUE TO COORDINATED EFFORTS 3668 02:17:31,000 --> 02:17:34,320 HAVING TO DO WITH THE PANDEMIC 3669 02:17:34,320 --> 02:17:36,960 MANY MANY MANY NIH WIDE 3670 02:17:36,960 --> 02:17:38,600 COLLABORATIVE PROJECTS INITIATED 3671 02:17:38,600 --> 02:17:45,720 IN FISCAL YEAR 2021. 3672 02:17:45,720 --> 02:17:47,280 SO I'M GOING TO EXPLAIN A LITTLE 3673 02:17:47,280 --> 02:17:48,760 BIT ABOUT INCLUSION ENROLLMENT 3674 02:17:48,760 --> 02:17:50,960 DATA, THE TABLES ON THE 3675 02:17:50,960 --> 02:17:55,240 FOLLOWING SLIDES SHOW INTERIM 3676 02:17:55,240 --> 02:17:55,960 CUMULATIVE DATA. 3677 02:17:55,960 --> 02:17:57,800 THEY ARE INTERIM BECAUSE THE 3678 02:17:57,800 --> 02:18:03,240 DATA REFLECT THE INFORMATION 3679 02:18:03,240 --> 02:18:04,560 COLLECTED BY NIH FOR STUDIES 3680 02:18:04,560 --> 02:18:06,240 THAT MAY CONTINUE PAST THE 3681 02:18:06,240 --> 02:18:07,800 SCHOOL YEAR 2021. 3682 02:18:07,800 --> 02:18:13,200 AND THEY ARE CUMULATIVE. 3683 02:18:13,200 --> 02:18:14,680 BECAUSE THEY COLLECT STUDIES 3684 02:18:14,680 --> 02:18:16,080 STARTED PRIOR TO FISCAL YEAR 3685 02:18:16,080 --> 02:18:18,000 2021. 3686 02:18:18,000 --> 02:18:20,640 SO THE FISCAL YEAR 19 AND 20 3687 02:18:20,640 --> 02:18:22,920 DATA ARE PROVIDED AS A BENCHMARK 3688 02:18:22,920 --> 02:18:27,760 FOR FISCAL YEAR 2021 DATA. 3689 02:18:27,760 --> 02:18:30,080 ALL STUDIES INVOLVING SECONDARY 3690 02:18:30,080 --> 02:18:32,120 DEALT ANALYSES ARE EXCLUDE -- 3691 02:18:32,120 --> 02:18:34,200 DATA ANALYSES ARE EXCLUDED. 3692 02:18:34,200 --> 02:18:35,880 THE DATA EXISTS PRIOR TO THE 3693 02:18:35,880 --> 02:18:39,600 STUDY, THEY ARE NOT INCLUDED IN 3694 02:18:39,600 --> 02:18:40,200 INCLUSION TABLES. 3695 02:18:40,200 --> 02:18:43,400 THE DATA SHOWING THE TABLES ARE 3696 02:18:43,400 --> 02:18:45,720 STANDARDIZED AND ALL REPRESENT 3697 02:18:45,720 --> 02:18:47,480 RESPONSES TO SPECIFIC REQUESTS 3698 02:18:47,480 --> 02:18:51,840 MADE TO EACH. 3699 02:18:51,840 --> 02:18:53,520 SO NOT DIRECTLY COMPARABLE. 3700 02:18:53,520 --> 02:18:54,960 FINALLY INCLUSION ENROLLMENT 3701 02:18:54,960 --> 02:18:59,040 DATA BY RESEARCH CONDITIONS AND 3702 02:18:59,040 --> 02:19:03,000 DISEASE CATEGORIZATION RCDC ARE 3703 02:19:03,000 --> 02:19:04,360 AVAILABLE THROUGH THE LINK 3704 02:19:04,360 --> 02:19:06,600 PROVIDED HERE. 3705 02:19:06,600 --> 02:19:12,160 NEXT SLIDE PLEASE. 3706 02:19:12,160 --> 02:19:14,360 DATA TABLES I WILL SHOW YOU, 3707 02:19:14,360 --> 02:19:15,560 THERE ARE NOT TOO MANY BUT I 3708 02:19:15,560 --> 02:19:17,880 WILL SHOW YOU DOMESTIC VERSUS 3709 02:19:17,880 --> 02:19:19,320 FOREIGN INCLUSION ENROLLMENT 3710 02:19:19,320 --> 02:19:20,480 TABLES. 3711 02:19:20,480 --> 02:19:23,280 NIH DEFINED PHASE 3 CLINICAL 3712 02:19:23,280 --> 02:19:24,200 TRIALS. 3713 02:19:24,200 --> 02:19:26,760 ENROLLMENT BY SEX GENDER, 3714 02:19:26,760 --> 02:19:29,640 ENROLLMENT BY SEX GENDER AND 3715 02:19:29,640 --> 02:19:31,600 RACE ETHNICITY. 3716 02:19:31,600 --> 02:19:34,320 U.S. MINORITY ENROLLMENT. 3717 02:19:34,320 --> 02:19:37,960 ENROLLMENT BY MINORITY RACE, AND 3718 02:19:37,960 --> 02:19:43,720 U.S. ENROLLMENT BY ETHNICITY. 3719 02:19:43,720 --> 02:19:45,320 WE WILL GET TO DATA TABLES. 3720 02:19:45,320 --> 02:19:50,800 NEXT SLIDE PLEASE. 3721 02:19:50,800 --> 02:19:52,600 THIS TABLE SHOAS THERE WERE MANY 3722 02:19:52,600 --> 02:19:54,800 MORE STUDIES CONDUCTED IN THE 3723 02:19:54,800 --> 02:19:56,400 U.S. THAN ABROAD. 3724 02:19:56,400 --> 02:20:03,760 THAT'S IN THE PURPLE BOX. 3725 02:20:03,760 --> 02:20:06,400 WHEN REPORT NUMBER OF INCLUSION 3726 02:20:06,400 --> 02:20:08,840 REPORTS IF 2021 WE ARE CLOSE TO 3727 02:20:08,840 --> 02:20:10,800 NUMBER IN 2019 THERE WERE FEW 3728 02:20:10,800 --> 02:20:12,120 MORE INCLUSION ENROLLMENT 3729 02:20:12,120 --> 02:20:14,080 REPORTS THAT DID NOT REPORT 3730 02:20:14,080 --> 02:20:16,560 ENROLLMENT AND THAT IS BECAUSE 3731 02:20:16,560 --> 02:20:18,760 NUMBER OF STUDIES HAD TO DELAY 3732 02:20:18,760 --> 02:20:22,800 ENROLLMENT DUE TO COVID. 3733 02:20:22,800 --> 02:20:25,160 SOME SIMPLY HAD NOT YET STARTED. 3734 02:20:25,160 --> 02:20:30,880 APOLOGIES BUT -- SO THE LOWER 3735 02:20:30,880 --> 02:20:32,760 TABLE INDICATES N CATS DIDN'T 3736 02:20:32,760 --> 02:20:34,440 SUPPORT NIH DEFINED PHASE 3 3737 02:20:34,440 --> 02:20:39,320 CLINICAL TRIALS IN 2021, WE NEED 3738 02:20:39,320 --> 02:20:43,000 TO GO THROUGH SPECIAL PROSESES 3739 02:20:43,000 --> 02:20:45,320 RARE DISEASES IF WE WANT TO DO 3740 02:20:45,320 --> 02:20:48,760 THIS TYPE OF STUDY. 3741 02:20:48,760 --> 02:20:51,160 SO WE DON'T HAVE VERY MANY YOU 3742 02:20:51,160 --> 02:20:54,960 CAN SEE PRIOR YEARS, AND NONE IN 3743 02:20:54,960 --> 02:21:03,680 FY 20216789 SO THESE THIS TABLE 3744 02:21:03,680 --> 02:21:05,000 SHOWS AGGREGATE ENROLLMENT BY 3745 02:21:05,000 --> 02:21:09,160 SEX GENDER, WHAT I WANT TO POINT 3746 02:21:09,160 --> 02:21:12,160 OUT IS THAT THE NUMBER OF 3747 02:21:12,160 --> 02:21:16,480 UNKNOWNS WHICH IS PURPLE ARROW, 3748 02:21:16,480 --> 02:21:18,280 NUMBER OF FIRST NUMBER OF 3749 02:21:18,280 --> 02:21:21,360 FEMALES ENROLLED IN 2021 IS 3750 02:21:21,360 --> 02:21:23,440 SIMILAR TO THE NUMBER OF MALES 3751 02:21:23,440 --> 02:21:23,960 ENROLLED. 3752 02:21:23,960 --> 02:21:26,240 THAT IS WHERE THE DATABASE GOT 3753 02:21:26,240 --> 02:21:26,760 UPDATED. 3754 02:21:26,760 --> 02:21:28,720 SO THAT IF IT LOOKS LARGER IT IS 3755 02:21:28,720 --> 02:21:30,520 NOT REALLY LARGER. 3756 02:21:30,520 --> 02:21:31,960 BUT I WANTED TO FOCUS YOUR 3757 02:21:31,960 --> 02:21:33,720 ATTENTION ON THE PERCENT OF 3758 02:21:33,720 --> 02:21:35,720 UNKNOWN ENROW LEES WHICH 3759 02:21:35,720 --> 02:21:36,960 DECREASED SIGNIFICANTLY IN 3760 02:21:36,960 --> 02:21:42,000 FISCAL YEAR 2021, DUE TO FOCUSED 3761 02:21:42,000 --> 02:21:43,160 EFFORT BY DIVISION OF CLINICAL 3762 02:21:43,160 --> 02:21:46,000 INNOVATION. 3763 02:21:46,000 --> 02:21:47,680 AND WE HAVE NOW REACHED THE 3764 02:21:47,680 --> 02:21:51,120 LEVEL THAT NIH GENERALLY HAS 3765 02:21:51,120 --> 02:21:55,800 OVERALL FOR UNKNOWNS AROUND 3% 3766 02:21:55,800 --> 02:21:57,160 SO WE HAVE MADE A LOT OF 3767 02:21:57,160 --> 02:22:05,480 PROGRESS IN THAT AREA. 3768 02:22:05,480 --> 02:22:07,360 NEXT SLIDE PLEASE. 3769 02:22:07,360 --> 02:22:10,120 THIS LARGE TABLE SHOWS DATA BY 3770 02:22:10,120 --> 02:22:11,920 SEX GENDER AND RACE ETHNICITY. 3771 02:22:11,920 --> 02:22:15,400 YOU CAN LOOK AT THIS TABLE AT 3772 02:22:15,400 --> 02:22:17,080 YOUR LEISURE BUT THE TAKE HOME 3773 02:22:17,080 --> 02:22:19,120 MESSAGES ARE THE NUMBER SO YOU 3774 02:22:19,120 --> 02:22:22,440 WILL SEE 2021 ARE THE LAST THREE 3775 02:22:22,440 --> 02:22:23,120 ROWS. 3776 02:22:23,120 --> 02:22:25,560 THE NUMBER OF FEMALE AND MALE 3777 02:22:25,560 --> 02:22:26,880 MINORITY ENROLLEES ARE SIMILAR 3778 02:22:26,880 --> 02:22:32,080 EACH FISCAL YEAR. 3779 02:22:32,080 --> 02:22:38,000 MOST IF FISCAL YEAR 2021, AS THE 3780 02:22:38,000 --> 02:22:40,560 SECOND BOX SHOWS THEY WERE WHITE 3781 02:22:40,560 --> 02:22:42,680 AND NON-HISPANIC. 3782 02:22:42,680 --> 02:22:45,600 THIS ISN'T SO UNUSUAL BUT I NEED 3783 02:22:45,600 --> 02:22:47,600 TO POINT OUT AND I WILL ADDRESS 3784 02:22:47,600 --> 02:22:49,600 THAT, IN A MINUTE. 3785 02:22:49,600 --> 02:22:55,160 NEXT SLIDE PLEASE. 3786 02:22:55,160 --> 02:22:57,120 THIS TABLE SHOWS PERCENT 3787 02:22:57,120 --> 02:23:00,000 MINORITY ENROLLMENT FOR FISCAL 3788 02:23:00,000 --> 02:23:01,960 YEAR 2021 IN THE UNITED STATES 3789 02:23:01,960 --> 02:23:04,840 IS QUITE SIMILAR ACROSS THE LAST 3790 02:23:04,840 --> 02:23:06,440 THREE YEARS SO LOOK AT THE 3791 02:23:06,440 --> 02:23:09,600 PERCENT MINORITY ENROLLEES IT 3792 02:23:09,600 --> 02:23:13,360 ISN'T VERY DIFFERENT, BUT 3793 02:23:13,360 --> 02:23:15,000 PERCENT MINORITY ENROLLEES IN 3794 02:23:15,000 --> 02:23:18,920 THE UNITED STATES WAS LOWER IN 3795 02:23:18,920 --> 02:23:19,920 2021. 3796 02:23:19,920 --> 02:23:22,680 SHOWN ON PREVIOUS SLIDE. 3797 02:23:22,680 --> 02:23:26,240 NEXT SLIDE PLEASE. 3798 02:23:26,240 --> 02:23:28,760 SO NOW WE HAVE ENROLLMENT BY 3799 02:23:28,760 --> 02:23:33,160 RACE WHICH IS ANOTHER TABLE BUT 3800 02:23:33,160 --> 02:23:34,320 WHAT I WANT YOU TO TAKE FROM 3801 02:23:34,320 --> 02:23:38,280 THIS IS THAT FISCAL YEAR 2021 3802 02:23:38,280 --> 02:23:39,880 SHOWS INCREASING PERCENTAGE OF 3803 02:23:39,880 --> 02:23:43,680 WHITE AND MORE THAN ONE RACE 3804 02:23:43,680 --> 02:23:44,360 ENROLLEES. 3805 02:23:44,360 --> 02:23:50,160 THESE DATA ARE SELF-REPORTED SO 3806 02:23:50,160 --> 02:23:51,960 THIS IS WHAT PARTICIPANTS 3807 02:23:51,960 --> 02:23:52,920 REPORTED. 3808 02:23:52,920 --> 02:23:54,840 THE PERCENTAGE OF ENROLLEES 3809 02:23:54,840 --> 02:23:56,200 GREATER THAN ONE RACE INCREASED 3810 02:23:56,200 --> 02:23:59,320 OVER THE LAST THREE YEARS. 3811 02:23:59,320 --> 02:24:01,520 AND PERCENTAGE ENROLLEES WITH 3812 02:24:01,520 --> 02:24:02,920 UNKNOWN RACE DECREASED THE LAST 3813 02:24:02,920 --> 02:24:06,000 THREE YEARS. 3814 02:24:06,000 --> 02:24:09,280 AS SHOWN BY ARROW ON THE RIGHT 3815 02:24:09,280 --> 02:24:10,360 SIDE. 3816 02:24:10,360 --> 02:24:17,280 NEXT SLIDE PLEASE. 3817 02:24:17,280 --> 02:24:18,240 ENROLLMENT BY ETHNICITY. 3818 02:24:18,240 --> 02:24:21,000 WHAT IT SHOWS IS PERCENTAGE OF 3819 02:24:21,000 --> 02:24:22,560 HISPANIC ENROLLEES DECREASED 3820 02:24:22,560 --> 02:24:26,600 BETWEEN FISCAL YEAR 2019 AND 3821 02:24:26,600 --> 02:24:27,160 2021. 3822 02:24:27,160 --> 02:24:33,880 NOT A LOT BUT SOME. 3823 02:24:33,880 --> 02:24:38,600 HA IS WHAT WE WILL ADDRESS GOING 3824 02:24:38,600 --> 02:24:39,560 FORWARD. 3825 02:24:39,560 --> 02:24:41,040 NEXT SLIDE PLEASE. 3826 02:24:41,040 --> 02:24:42,040 NCATS SUPPORTS EFFORTS TO 3827 02:24:42,040 --> 02:24:44,480 INCREASE MINORITY PARTICIPATION 3828 02:24:44,480 --> 02:24:47,160 AND DIVERSITY. 3829 02:24:47,160 --> 02:24:48,760 NCATS SPECIFIC PROGRAMS AND IN 3830 02:24:48,760 --> 02:24:50,640 NIH PROGRAMS THAT LEVERAGE NCATS 3831 02:24:50,640 --> 02:24:52,960 PROGRAMS AND RESOURCES. 3832 02:24:52,960 --> 02:24:54,880 WE RECOGNIZE THE RELATIONSHIP 3833 02:24:54,880 --> 02:24:57,720 BETWEEN DIVERSITY AND INCLUSION 3834 02:24:57,720 --> 02:25:00,160 AND RESEARCH FUNDING TRAINING 3835 02:25:00,160 --> 02:25:02,920 RESEARCH PARTICIPATION ACCESS TO 3836 02:25:02,920 --> 02:25:04,600 RESEARCH BENEFIT HEALTHCARE AND 3837 02:25:04,600 --> 02:25:06,720 POPULATION HEALTH. 3838 02:25:06,720 --> 02:25:08,960 THE GOAL OF NCATS TRANSLATIONAL 3839 02:25:08,960 --> 02:25:10,880 SCIENCE IS TO FACILITATE 3840 02:25:10,880 --> 02:25:13,400 EQUITABLE BENEFIT TO ALL 3841 02:25:13,400 --> 02:25:15,840 REGARDLESS OF RACE ETHNICITY 3842 02:25:15,840 --> 02:25:20,000 SEXUAL O ORIENTATION, 3843 02:25:20,000 --> 02:25:22,000 SOCIOECONOMIC IS A TUS OR 3844 02:25:22,000 --> 02:25:23,080 BACKGROUND, WE WANT TO 3845 02:25:23,080 --> 02:25:24,760 STRENGTHEN PARTNERSHIPS WITH 3846 02:25:24,760 --> 02:25:26,600 COLLABORATORS TO ENSURE THAT 3847 02:25:26,600 --> 02:25:29,520 EQUITY DIVERSITY AND INCLUSION 3848 02:25:29,520 --> 02:25:30,920 ARE MEANINGFULLY ADDRESSED AT 3849 02:25:30,920 --> 02:25:33,960 ALL NCATS INITIATIVES. 3850 02:25:33,960 --> 02:25:36,160 AND WE TEND TO PROVIDE PUBLIC 3851 02:25:36,160 --> 02:25:37,840 TRANSPARENCY ON THE EQUITY 3852 02:25:37,840 --> 02:25:40,840 DIVERSITY ANDEN COLLUSION GOALS. 3853 02:25:40,840 --> 02:25:42,760 ACTION AND METRICS OF NCATS 3854 02:25:42,760 --> 02:25:45,800 RESEARCH PROJECT AND ACTIVITIES. 3855 02:25:45,800 --> 02:25:48,360 INCLUDING GRANTS, INTRAMURAL 3856 02:25:48,360 --> 02:25:50,000 PROJECTS TRAINING PROGRAMS 3857 02:25:50,000 --> 02:25:51,960 WORKSHOPS, COLLABORATIONS, AND 3858 02:25:51,960 --> 02:25:56,160 AREAS OF TRANSLATIONAL FOCUS. 3859 02:25:56,160 --> 02:26:00,440 AND THAT IS ALL I'M GOING TO 3860 02:26:00,440 --> 02:26:02,000 PRESENT TO YOU TODAY BUT I'M 3861 02:26:02,000 --> 02:26:04,880 HAPPY TO ANSWER QUESTIONS 3862 02:26:04,880 --> 02:26:05,720 PARTICIPATE IN DISCUSSION. 3863 02:26:05,720 --> 02:26:06,560 THANK YOU. 3864 02:26:06,560 --> 02:26:08,560 >> THANK YOU SO MUCH, VALERY. 3865 02:26:08,560 --> 02:26:11,880 I WILL REITERATE REMINDER THIS 3866 02:26:11,880 --> 02:26:12,960 IS SOMETHING THAT IS REQUIRED 3867 02:26:12,960 --> 02:26:15,240 FOR US TO DO TO PRESENT THIS TO 3868 02:26:15,240 --> 02:26:16,320 COUNCIL. 3869 02:26:16,320 --> 02:26:19,360 AND YOU ALSO HAVE THE MUCH 3870 02:26:19,360 --> 02:26:22,080 DEEPER SET OF INFORMATION IN 3871 02:26:22,080 --> 02:26:23,280 YOUR COUNCIL BOOKS SO YOU CAN 3872 02:26:23,280 --> 02:26:25,920 REVIEW THEM AND ANOTHER 3873 02:26:25,920 --> 02:26:27,560 IMPORTANT ASPECT OF THIS IS THAT 3874 02:26:27,560 --> 02:26:29,200 I ALSO GO THROUGH AND CERTIFY 3875 02:26:29,200 --> 02:26:31,160 THESE NUMBERS AS WELL TO SEND 3876 02:26:31,160 --> 02:26:36,000 THEM TO NIH AND AS THAT BIGGER 3877 02:26:36,000 --> 02:26:38,000 ALL OF NIH REPORT GETS PUT 3878 02:26:38,000 --> 02:26:39,440 TOGETHER AND SO UNDERSTANDING 3879 02:26:39,440 --> 02:26:41,200 THE IMPACTS OF WHAT NIH 3880 02:26:41,200 --> 02:26:42,880 CONTRIBUTES TO THESE NUMBERS AS 3881 02:26:42,880 --> 02:26:44,400 WELL AS THEN UNDERSTANDING WHAT 3882 02:26:44,400 --> 02:26:46,360 IT IS IN THE LARGER PICTURE WHEN 3883 02:26:46,360 --> 02:26:47,760 WE SEE THAT FINAL REPORT IS 3884 02:26:47,760 --> 02:26:48,160 IMPORTANT. 3885 02:26:48,160 --> 02:26:49,520 SO WE WILL BE LOOKING AT THAT 3886 02:26:49,520 --> 02:26:51,960 WHEN IT COMES OUT TOO. 3887 02:26:51,960 --> 02:26:53,920 THESE WERE NCATS NUMBERS I WANT 3888 02:26:53,920 --> 02:26:56,440 TO GIVE YOU AN OPPORTUNITY TO 3889 02:26:56,440 --> 02:26:57,640 ASK ANY QUESTIONS YOU MIGHT HAVE 3890 02:26:57,640 --> 02:27:00,800 IN TERMS OF THIS REPORT SO I SEE 3891 02:27:00,800 --> 02:27:02,280 PAULA AND MARSHALL HAVE YOUR 3892 02:27:02,280 --> 02:27:04,440 HAND UP, PAULA YOU GO FIRST. 3893 02:27:04,440 --> 02:27:09,720 >> THANKS, IT WAS A VERY 3894 02:27:09,720 --> 02:27:10,280 ILLUMINATING PRESENTATION. 3895 02:27:10,280 --> 02:27:12,720 ANOTHER THING I FIND SURPRISING 3896 02:27:12,720 --> 02:27:15,120 IS HITS PANIC ENROLLMENT WENT 3897 02:27:15,120 --> 02:27:17,880 DOWN WHEN HISPANIC ETHNICITY IS 3898 02:27:17,880 --> 02:27:19,640 THE MORE RAPIDLY GROWING 3899 02:27:19,640 --> 02:27:21,280 SEGMENTS OF OUR POPULATION IN 3900 02:27:21,280 --> 02:27:24,080 THE UNITED STATES. 3901 02:27:24,080 --> 02:27:27,040 YOU ALSO NOTE THAT THE UNKNOWNS, 3902 02:27:27,040 --> 02:27:31,480 RACE AND ETHNICITY THE ALWAYS A 3903 02:27:31,480 --> 02:27:34,400 CHALLENGE WHEN IT COMES TO 3904 02:27:34,400 --> 02:27:35,480 LOOKING AT ANYTHING. 3905 02:27:35,480 --> 02:27:38,760 BECAUSE THE DATA IS OFTEN 3906 02:27:38,760 --> 02:27:40,640 MISSING WRONG INCORRECT 3907 02:27:40,640 --> 02:27:43,400 CONFLICTING AND EVERYTHING ELSE. 3908 02:27:43,400 --> 02:27:48,080 I GUESS ONE QUESTION IS HOW DO 3909 02:27:48,080 --> 02:27:50,280 WE IMPROVE THAT SO WE DON'T HAVE 3910 02:27:50,280 --> 02:27:52,560 A BIGGER UNKNOWN CATEGORY THAN 3911 02:27:52,560 --> 02:27:54,760 WE HAVE OF ANY SPECIFIC RACE OR 3912 02:27:54,760 --> 02:27:56,560 ETHNICITY. 3913 02:27:56,560 --> 02:27:58,200 SO HA IS ONE. 3914 02:27:58,200 --> 02:28:00,080 AND THE IDEA OF THE DIVERSITY 3915 02:28:00,080 --> 02:28:02,680 EQUITY AND INCLUSION IN WHAT WE 3916 02:28:02,680 --> 02:28:05,400 HAVE SEEN WITH LOW RESOURCE 3917 02:28:05,400 --> 02:28:08,040 AREAS LIKE RAWRL (INAUDIBLE) HOW 3918 02:28:08,040 --> 02:28:13,800 WE BEGIN TO ADD THOSE VARIABLES. 3919 02:28:13,800 --> 02:28:16,000 INTO THE MIX OF WHAT IS ALREADY 3920 02:28:16,000 --> 02:28:19,120 A VERY COMPLEX AREA AND TOPIC. 3921 02:28:19,120 --> 02:28:22,760 SO YES, I DO APPRECIATE THE 3922 02:28:22,760 --> 02:28:25,800 CHALLENGE YET WE HAVE TO 3923 02:28:25,800 --> 02:28:28,040 OVERCOME CHALLENGES (INAUDIBLE) 3924 02:28:28,040 --> 02:28:29,520 >> I COULDN'T AGREE WITH YOU 3925 02:28:29,520 --> 02:28:31,200 MORE. 3926 02:28:31,200 --> 02:28:35,240 WE WERE SURPRISED BY THE LOWER 3927 02:28:35,240 --> 02:28:40,760 HISPANIC ENROLLMENT AS I SAID, 3928 02:28:40,760 --> 02:28:43,600 THE LOW NUMBERS THAT WE HAVE 3929 02:28:43,600 --> 02:28:45,240 MAGNIFY THE DIFFERENCES. 3930 02:28:45,240 --> 02:28:49,360 SO I HOPE THAT YOU WON'T TAKE 3931 02:28:49,360 --> 02:28:52,960 THIS AS THAT WE ARE NOT PAYING 3932 02:28:52,960 --> 02:28:55,280 ATTENTION TO THIS BECAUSE WE ARE 3933 02:28:55,280 --> 02:28:58,520 PAYING ATTENTION TO THIS AND 3934 02:28:58,520 --> 02:29:03,120 THAT ANY HICCUPS MANY THE DATA, 3935 02:29:03,120 --> 02:29:04,320 THAT WILL BE CORRECTED LATER 3936 02:29:04,320 --> 02:29:05,760 STILL SHOW UP IN THE REPORT 3937 02:29:05,760 --> 02:29:10,520 BECAUSE IT IS ONLY RUN ONCE. 3938 02:29:10,520 --> 02:29:11,960 SO I CAN'T PROMISE EXRU THESE 3939 02:29:11,960 --> 02:29:14,360 DATA ARE ABSOLUTELY CORRECT. 3940 02:29:14,360 --> 02:29:15,560 I CAN PROMISE YOU THAT THAT'S 3941 02:29:15,560 --> 02:29:18,920 WHAT WAS IN THE DATABASE ON THE 3942 02:29:18,920 --> 02:29:20,000 -- THAT THE DAY THE REPORT WAS 3943 02:29:20,000 --> 02:29:22,240 RUN. 3944 02:29:22,240 --> 02:29:26,160 IN ANY CASE WE ARE STILL TRYING 3945 02:29:26,160 --> 02:29:29,160 TO -- WE HAVE A GOAL TO ENSURE 3946 02:29:29,160 --> 02:29:30,640 THAT EQUITY DIVERSITY AND 3947 02:29:30,640 --> 02:29:32,200 INCLUSION ARE MEANINGFULLY 3948 02:29:32,200 --> 02:29:41,080 ADDRESSED BY N CATS. 3949 02:29:41,080 --> 02:29:45,760 >> VALERY, I WILL ADD AS WELL, 3950 02:29:45,760 --> 02:29:47,840 THESE NUMBERS ARE DYNAMIC SO 3951 02:29:47,840 --> 02:29:51,760 WHEN YOU LOOK AT THEM, THEY ARE 3952 02:29:51,760 --> 02:29:52,720 DIFFICULT TO INTERPRET BECAUSE 3953 02:29:52,720 --> 02:29:56,680 THEY ARE SO DYNAMIC, SO WE DON'T 3954 02:29:56,680 --> 02:29:58,440 WANT TO OVERREACT BUT SOMETHING 3955 02:29:58,440 --> 02:30:00,680 LIKE THAT YOU POINTED OUT, I 3956 02:30:00,680 --> 02:30:02,560 THINK OVER REACTION IS NOT BAD 3957 02:30:02,560 --> 02:30:02,920 THING. 3958 02:30:02,920 --> 02:30:03,960 SOME OF THE THINGS WE ARE 3959 02:30:03,960 --> 02:30:08,760 WORKING ON TOO FOR EXAMPLE ARE 3960 02:30:08,760 --> 02:30:12,640 DISCUSSIONS WITH NIMHD IN 3961 02:30:12,640 --> 02:30:14,840 TALKING ABOUT THEIR RCMI 3962 02:30:14,840 --> 02:30:17,080 INSTITUTIONS HOW WE CAN FIND 3963 02:30:17,080 --> 02:30:18,560 COORDINATION COLLABORATION. 3964 02:30:18,560 --> 02:30:20,120 SO EVEN THOUGH THOSE NUMBERS 3965 02:30:20,120 --> 02:30:22,960 MIGHT BE DYNAMIC AND THAT IS 3966 02:30:22,960 --> 02:30:24,840 IMPORTANT TO KNOW THERE'S STILL 3967 02:30:24,840 --> 02:30:26,520 -- THEY STILL HIGHLIGHT SOME 3968 02:30:26,520 --> 02:30:28,600 ISSUES WE STILL NEED TO ADDRESS 3969 02:30:28,600 --> 02:30:30,480 AND THAT IS ONE OF THOSE THAT 3970 02:30:30,480 --> 02:30:32,080 YOU POINT OUT THAT I THINK WE 3971 02:30:32,080 --> 02:30:32,680 CAN. 3972 02:30:32,680 --> 02:30:34,080 SO JUST SOME OF THOSE THINGS WE 3973 02:30:34,080 --> 02:30:38,920 ARE TRYING TO DO ARE ENCOURAGING 3974 02:30:38,920 --> 02:30:40,120 MORE INTERACTION ACROSS 3975 02:30:40,120 --> 02:30:42,360 INSTITUTIONS, THE CTSA PROGRAM 3976 02:30:42,360 --> 02:30:45,040 IS FOR EXAMPLE MANY THE COVID 3977 02:30:45,040 --> 02:30:45,960 ENVIRONMENT THEY HAVE BEEN 3978 02:30:45,960 --> 02:30:49,120 WORKING WITH NHLBI FOR THIS SEAL 3979 02:30:49,120 --> 02:30:50,480 INITIATIVE IN TERMS OF DOING 3980 02:30:50,480 --> 02:30:52,480 MORE IN TERMS OF ENGAGEMENT. 3981 02:30:52,480 --> 02:30:54,760 AND COMMUNICATION ABOUT COVID 3982 02:30:54,760 --> 02:30:55,480 ACTIVITIES. 3983 02:30:55,480 --> 02:30:59,240 AND THAT IS REALLY ENHANCED THE 3984 02:30:59,240 --> 02:31:01,240 AWARNESS OF HOW PEOPLE GET 3985 02:31:01,240 --> 02:31:03,000 INVOLVED IN THESE TYPES OF 3986 02:31:03,000 --> 02:31:03,240 STUDIES. 3987 02:31:03,240 --> 02:31:04,960 THAT IS ONE ASPECT WE CAN TRY TO 3988 02:31:04,960 --> 02:31:09,440 CONTINUE TO PROMOTE AND THOSE 3989 02:31:09,440 --> 02:31:11,320 INTERACTIONS. 3990 02:31:11,320 --> 02:31:16,080 I WANT TO REITERATE WHAT VALERY 3991 02:31:16,080 --> 02:31:17,840 SAID, THOUGH SYSTEM ISN'T 3992 02:31:17,840 --> 02:31:18,760 PERFECT IT IS STILL SOMETHING WE 3993 02:31:18,760 --> 02:31:21,400 CAN REALLY HANG OUR HAT ON AND 3994 02:31:21,400 --> 02:31:23,800 TRY TO BOLSTER WORK IN THIS 3995 02:31:23,800 --> 02:31:24,320 SPACE. 3996 02:31:24,320 --> 02:31:27,120 >> JONI, LET ME JUST ADD THAT AS 3997 02:31:27,120 --> 02:31:28,960 VALERY POINTED OUT, HOPEFULLY IT 3998 02:31:28,960 --> 02:31:32,560 DIDN'T GET LOST, SOME LARGE 3999 02:31:32,560 --> 02:31:34,960 EFFORTS AT NCATS IS INVOLVED 4000 02:31:34,960 --> 02:31:40,880 WITH ACTIONS SUCH AS HING THINGS LIKE 4001 02:31:40,880 --> 02:31:43,680 SEAL, NHLBI RUN THE TRIAL 4002 02:31:43,680 --> 02:31:45,080 INNOVATION NETWORK, THAT'S NOT 4003 02:31:45,080 --> 02:31:46,520 PART OF THIS REPORTING BECAUSE 4004 02:31:46,520 --> 02:31:48,360 THAT IS REPORTED ELSEWHERE SO 4005 02:31:48,360 --> 02:31:52,920 WILL IS A LOT OF ENGAGEMENT BY 4006 02:31:52,920 --> 02:31:54,720 NCATS BUT OTHER PEOPLE ARE 4007 02:31:54,720 --> 02:31:56,520 REPORTING THOSE NUMBERS. 4008 02:31:56,520 --> 02:32:00,800 SO OURS IS A VERY SMALL SUBSET 4009 02:32:00,800 --> 02:32:05,960 OF THE TOTAL ACTIVITIES THAT WE 4010 02:32:05,960 --> 02:32:08,560 ARE ENGAGED WITH AND TENDS TO BE 4011 02:32:08,560 --> 02:32:10,720 FOCUSED ON SMALLER PILOT SCALE 4012 02:32:10,720 --> 02:32:11,600 PROJECTS WITH YOUNGER 4013 02:32:11,600 --> 02:32:12,440 INVESTIGATORS. 4014 02:32:12,440 --> 02:32:13,960 SO IT IS SOMETHING WE PUT A LOT 4015 02:32:13,960 --> 02:32:16,120 OF EFFORT INTO WORKING MORE 4016 02:32:16,120 --> 02:32:16,960 CLOSELY WITH. 4017 02:32:16,960 --> 02:32:19,200 >> AND AGAIN EVEN SO, IT IS 4018 02:32:19,200 --> 02:32:21,960 STILL SOMETHING WE WANT TO 4019 02:32:21,960 --> 02:32:23,200 RECOGNIZE TO ADDRESS FURTHER. 4020 02:32:23,200 --> 02:32:25,200 PAUL I SEE YOUR HAND UP NEXT. 4021 02:32:25,200 --> 02:32:26,040 PLEASE GO AHEAD. 4022 02:32:26,040 --> 02:32:27,720 >> THANK YOU FOR THE 4023 02:32:27,720 --> 02:32:28,400 PRESENTATION. 4024 02:32:28,400 --> 02:32:29,600 SOME OF THE NUMBERS WERE A 4025 02:32:29,600 --> 02:32:31,560 LITTLE DISTURBING BUT I DO 4026 02:32:31,560 --> 02:32:35,000 UNDERSTAND THEIR FLUID. 4027 02:32:35,000 --> 02:32:37,840 THERE IS ALWAYS ROOM FOR 4028 02:32:37,840 --> 02:32:40,280 IMPROVEMENT SO I APPRECIATE YOU 4029 02:32:40,280 --> 02:32:41,520 GOING DEEPER THAN THE LAST SLIDE 4030 02:32:41,520 --> 02:32:43,160 IN TERMS OF THE TACTICS FOR THE 4031 02:32:43,160 --> 02:32:47,360 REMEDIATION PLAN. 4032 02:32:47,360 --> 02:32:49,560 AS THE EAC IS IT APPROPRIATE TO 4033 02:32:49,560 --> 02:32:51,400 ASK FOR UPDATE NEXT TIME WE 4034 02:32:51,400 --> 02:32:54,160 MEET? 4035 02:32:54,160 --> 02:32:58,560 MAINLY DETAILING SOME OF THE 4036 02:32:58,560 --> 02:32:59,880 STRATEGIES AND TACTICS UNDERWAY 4037 02:32:59,880 --> 02:33:00,400 THERE? 4038 02:33:00,400 --> 02:33:02,800 >> HAPPY TO DO THAT. 4039 02:33:02,800 --> 02:33:03,520 >> THANKS. 4040 02:33:03,520 --> 02:33:04,240 >> GOOD SUGGESTION. 4041 02:33:04,240 --> 02:33:04,640 THANK YOU. 4042 02:33:04,640 --> 02:33:08,000 >> WE GENERATE INTERNAL REPORT 4043 02:33:08,000 --> 02:33:09,840 ANNUALLY. 4044 02:33:09,840 --> 02:33:11,360 SO THAT -- THOSE NUMBERS WILL BE 4045 02:33:11,360 --> 02:33:15,120 AVAILABLE TO YOU. 4046 02:33:15,120 --> 02:33:15,960 >> OKAY. 4047 02:33:15,960 --> 02:33:18,240 ANY OTHER -- I SEE MARSHALL YOUR 4048 02:33:18,240 --> 02:33:19,560 HAND UP MAYBE THE LAST WORD 4049 02:33:19,560 --> 02:33:21,280 HERE. 4050 02:33:21,280 --> 02:33:24,520 >> THAT'S TWICE NOW. 4051 02:33:24,520 --> 02:33:26,080 TWO SHORT OBSERVATIONS. 4052 02:33:26,080 --> 02:33:27,520 ONE VALERY, ONE OF THE THINGS WE 4053 02:33:27,520 --> 02:33:29,960 SEE IN OUR STATIC POPULATION 4054 02:33:29,960 --> 02:33:31,560 CLINICS AT LEAST THE CHILDREN'S 4055 02:33:31,560 --> 02:33:34,160 HOSPITAL IS FAMILY MORE 4056 02:33:34,160 --> 02:33:35,120 FREQUENTLY IDENTIFYING MORE THAN 4057 02:33:35,120 --> 02:33:37,240 ONE RACE. 4058 02:33:37,240 --> 02:33:40,480 SO WE HAVE SEEN A BIG SHIFT 4059 02:33:40,480 --> 02:33:41,760 WITHOUT NECESSARILY SHIFT WHO 4060 02:33:41,760 --> 02:33:42,840 THE ACTUAL PATIENTS ARE SO THAT 4061 02:33:42,840 --> 02:33:47,760 DOES NOT SURPRISE ME. 4062 02:33:47,760 --> 02:33:49,320 THAT IS CONSISTENT WITH OTHER 4063 02:33:49,320 --> 02:33:50,720 TRENDS ELSEWHERE. 4064 02:33:50,720 --> 02:33:52,000 WHEN WE WENT BACK AND 4065 02:33:52,000 --> 02:33:53,600 INTERVIEWED PEOPLE WHO CHOSE NOT 4066 02:33:53,600 --> 02:33:56,960 TO PARTICIPATE IN RESEARCH FROM 4067 02:33:56,960 --> 02:33:59,880 DIVERSE OR FROM OUR HISPANIC 4068 02:33:59,880 --> 02:34:01,280 NON-ENGLISH SPEAKING COMMUNITIES 4069 02:34:01,280 --> 02:34:05,640 ONE BIG IDENTIFIER WAS THE IRB. 4070 02:34:05,640 --> 02:34:08,960 PEOPLE FELT THREATENED, VERY 4071 02:34:08,960 --> 02:34:11,640 OVERWHELMED, IT IS LIKE THAT 4072 02:34:11,640 --> 02:34:13,720 APPLE AGREE THING WHERE YOU LOOK 4073 02:34:13,720 --> 02:34:16,160 AND SAY WHAT DID I AGREE TO ONLY 4074 02:34:16,160 --> 02:34:20,400 MORE SIGNIFICANT HERE. 4075 02:34:20,400 --> 02:34:23,280 SO ONE AREA YOU CAN LOOK AT THAT 4076 02:34:23,280 --> 02:34:26,000 IS ISSUES AROUNDISHRB AND 4077 02:34:26,000 --> 02:34:27,200 CONSENT. 4078 02:34:27,200 --> 02:34:29,200 >> A MORE OBVIOUS STATEMENT, THE 4079 02:34:29,200 --> 02:34:30,560 ELEPHANT IN THE ROOM IS THE 4080 02:34:30,560 --> 02:34:33,840 PANDEMIC CLEARLY HAS AFFECTED 4081 02:34:33,840 --> 02:34:39,280 OUR SOCIETY UNEQUALLY AND SHOWN 4082 02:34:39,280 --> 02:34:42,080 CHALLENGES. 4083 02:34:42,080 --> 02:34:44,560 WHAT YOU SEE IN NUMBERS IN THE 4084 02:34:44,560 --> 02:34:46,160 SLEKS OF THAT AND THAT'S BACK TO 4085 02:34:46,160 --> 02:34:48,920 PAUL'S COMMENT WE HAVE TO ENSURE 4086 02:34:48,920 --> 02:34:52,000 THAT THAT IS A CONSEQUENCE OF 4087 02:34:52,000 --> 02:34:53,000 EXTERNAL FORCE WE ARE UNDER 4088 02:34:53,000 --> 02:34:56,760 RATHER THE THAN INTRINSIC THAT 4089 02:34:56,760 --> 02:34:58,640 MIGHT BE A REASONABLE WAY TO 4090 02:34:58,640 --> 02:35:00,080 FOLLOW HOW CLOSE WE PARTICULARLY 4091 02:35:00,080 --> 02:35:01,960 OUR MINORITY POPULATIONS RECOVER 4092 02:35:01,960 --> 02:35:05,720 FROM THE PANDEMIC. 4093 02:35:05,720 --> 02:35:08,560 WE TAKE AS AN OPPORTUNITY TO 4094 02:35:08,560 --> 02:35:08,960 ENGAGEMENT. 4095 02:35:08,960 --> 02:35:10,640 >> THANK YOU, MATTHIAS. 4096 02:35:10,640 --> 02:35:13,440 WELL TAKEN. 4097 02:35:13,440 --> 02:35:15,080 ANY OTHER COMMENTS ON THIS 4098 02:35:15,080 --> 02:35:22,360 TOPIC? 4099 02:35:22,360 --> 02:35:24,920 FROM PAUL AND MATTHIAS, I WILL 4100 02:35:24,920 --> 02:35:26,440 MAKE SURE WE PRESENT THIS NEXT 4101 02:35:26,440 --> 02:35:29,000 TIME TO YOU TO GIVE A DEEPER 4102 02:35:29,000 --> 02:35:30,760 SENSE IN TERMS OF OUR MANAGING 4103 02:35:30,760 --> 02:35:33,120 THESE ISSUES. 4104 02:35:33,120 --> 02:35:35,360 I THINK WE WILL GO AHEAD AND 4105 02:35:35,360 --> 02:35:36,760 MOVE ON TO THE NEXT 4106 02:35:36,760 --> 02:35:37,360 PRESENTATION. 4107 02:35:37,360 --> 02:35:39,440 THIS IS THE LAST PRESENTATION OF 4108 02:35:39,440 --> 02:35:41,920 THE DAY. 4109 02:35:41,920 --> 02:35:43,880 BY KEITH LAMIRANDE, OUR 4110 02:35:43,880 --> 02:35:44,560 EXECUTIVE OFFICER. 4111 02:35:44,560 --> 02:35:46,520 HE DOESN'T OFTEN GET A CHANCE TO 4112 02:35:46,520 --> 02:35:47,720 PRESENT TO COUNCIL SO I'M 4113 02:35:47,720 --> 02:35:49,040 EXCITED, AND HE WILL BE TALKING 4114 02:35:49,040 --> 02:35:53,360 ABOUT SOME PROPOSED CHANGES THAT 4115 02:35:53,360 --> 02:35:54,800 REQUIRE PUBLIC PRESENTATION. 4116 02:35:54,800 --> 02:35:55,960 KEITH, I WILL TURN IT OVER TO 4117 02:35:55,960 --> 02:35:57,880 YOU. 4118 02:35:57,880 --> 02:35:59,520 >> GOOD AFTERNOON, EVERYBODY. 4119 02:35:59,520 --> 02:36:02,080 THANK YOU, JONI. 4120 02:36:02,080 --> 02:36:05,560 I'M EXECUTIVE OFFICER FOR NCATS. 4121 02:36:05,560 --> 02:36:07,080 THRILLED TO BE HERE WITH YOU 4122 02:36:07,080 --> 02:36:08,720 THIS AFTERNOON AS LAST THING 4123 02:36:08,720 --> 02:36:10,520 BETWEEN YOU AND NOT ONLY THE END 4124 02:36:10,520 --> 02:36:12,600 OF COUNCIL BUT THE WEEKEND. 4125 02:36:12,600 --> 02:36:15,800 SO WITH THE REFERENCE HERE LET 4126 02:36:15,800 --> 02:36:16,960 ME SAY MOW FOR SOMETHING 4127 02:36:16,960 --> 02:36:17,960 COMPLETELY DIFFERENT. 4128 02:36:17,960 --> 02:36:21,160 WE WILL TALK ABOUT 4129 02:36:21,160 --> 02:36:23,240 ORGANIZATIONAL CHANGES AND 4130 02:36:23,240 --> 02:36:24,720 SAUSAGE MAKING WITHIN THE 4131 02:36:24,720 --> 02:36:28,760 ORGANIZATIONAL STRUCTURE AT NIH. 4132 02:36:28,760 --> 02:36:30,520 I DON'T THINK I'M DRIVING THE 4133 02:36:30,520 --> 02:36:31,920 SLIDES SO I'LL SAY NEXT SLIDE 4134 02:36:31,920 --> 02:36:32,760 SEE WHAT HAPPENS. 4135 02:36:32,760 --> 02:36:34,120 PERFECT. 4136 02:36:34,120 --> 02:36:36,280 SO I'M GOING TO TALK AGAIN ABOUT 4137 02:36:36,280 --> 02:36:38,520 THE NIH REFORM ACT OF 2006. 4138 02:36:38,520 --> 02:36:41,040 IT WAS REFERENCED YESTERDAY WHEN 4139 02:36:41,040 --> 02:36:44,480 DR. SCHWETZ WAS TALKING AN'T 4140 02:36:44,480 --> 02:36:46,320 ARPA H AND THE LANGUAGE IN THAT 4141 02:36:46,320 --> 02:36:48,560 ACT THAT TALKS THE NEED FOR NO 4142 02:36:48,560 --> 02:36:49,960 MORE THAN 27 INSTITUTES AND 4143 02:36:49,960 --> 02:36:50,320 CENTERS. 4144 02:36:50,320 --> 02:36:53,960 IN ADDITION TO THAT LANGUAGE, 4145 02:36:53,960 --> 02:36:55,320 PART OF THE NIH REFORM ACT THERE 4146 02:36:55,320 --> 02:36:56,960 IS A DISCUSSION ABOUT THE 4147 02:36:56,960 --> 02:36:57,880 INTERNAL REORGANIZATION OF 4148 02:36:57,880 --> 02:37:00,880 INSTITUTES AND CENTERS. 4149 02:37:00,880 --> 02:37:02,640 THIS IS THE NEED FOR SERIES OF 4150 02:37:02,640 --> 02:37:04,520 PUBLIC HEARINGS AND THE APPROVAL 4151 02:37:04,520 --> 02:37:06,360 OF DIRECTOR OF NIH TO ALLOW US 4152 02:37:06,360 --> 02:37:08,560 TO REORGANIZE DIVISION CENTERS 4153 02:37:08,560 --> 02:37:11,040 AND OTHER ADMINISTRATIVE UNITS 4154 02:37:11,040 --> 02:37:11,840 WITHIN INSTITUTE OR CENTER. 4155 02:37:11,840 --> 02:37:15,280 THIS DOESN'T APPLY TO LOWER 4156 02:37:15,280 --> 02:37:17,160 LEVEL TYPES OF ORGANIZATIONAL 4157 02:37:17,160 --> 02:37:17,640 CHANGES. 4158 02:37:17,640 --> 02:37:20,280 WHICH MIGHT BE CREATING SECTIONS 4159 02:37:20,280 --> 02:37:21,600 UNDERNEATH EXISTING DIVISION OR 4160 02:37:21,600 --> 02:37:25,560 OFFICE BUT TO ELEVATE OR ADD 4161 02:37:25,560 --> 02:37:27,360 DIVISIONS TO ORGANIZAL STRUCTURE 4162 02:37:27,360 --> 02:37:29,040 INSTITUTE AN CENTER PARTICULARLY 4163 02:37:29,040 --> 02:37:30,280 ONE THAT ADDS REPORTING 4164 02:37:30,280 --> 02:37:31,520 STRUCTURE TO THE DIRECTOR 4165 02:37:31,520 --> 02:37:33,280 THEMSELVES. 4166 02:37:33,280 --> 02:37:35,080 WE ARE REQUIRED TO HAVE TWO 4167 02:37:35,080 --> 02:37:36,160 PUBLIC HEARINGS. 4168 02:37:36,160 --> 02:37:37,960 AND SINCE DWRAWL ARE KIND ENOUGH 4169 02:37:37,960 --> 02:37:40,720 TO HAVE COUNCIL AND OPEN COUNCIL 4170 02:37:40,720 --> 02:37:43,720 SESSION WE CHOSE TO JUMP ON IT 4171 02:37:43,720 --> 02:37:46,760 AND USE THIS AS ONE OF OUR TWO 4172 02:37:46,760 --> 02:37:47,200 PUBLIC HEARINGS. 4173 02:37:47,200 --> 02:37:48,160 THANK YOU VERY MUCH FOR THAT. 4174 02:37:48,160 --> 02:37:50,760 NEXT SLIDE PLEASE. 4175 02:37:50,760 --> 02:37:53,360 HERE YOU WILL SEE AS I THINK WE 4176 02:37:53,360 --> 02:37:56,240 STARTED OUR COUNCIL MEETING WITH 4177 02:37:56,240 --> 02:37:57,760 EXAMPLE OF ORGANIZATIONAL CHART 4178 02:37:57,760 --> 02:37:58,280 LOOKS LIKE. 4179 02:37:58,280 --> 02:37:59,800 THIS IS AN ORGANIZATIONAL CHART 4180 02:37:59,800 --> 02:38:02,160 THAT IDENTIFIES THE DIVISION OF 4181 02:38:02,160 --> 02:38:03,600 PRE-CLINICAL INNOVATION AND 4182 02:38:03,600 --> 02:38:04,920 DIVISION OF CLINICAL INNOVATION, 4183 02:38:04,920 --> 02:38:07,200 THESE ARE TWO DIVISIONS THAT 4184 02:38:07,200 --> 02:38:08,480 CURRENTLY EXIST WITHIN NCATS. 4185 02:38:08,480 --> 02:38:09,800 ALL THE OTHER REPORTING UNITS 4186 02:38:09,800 --> 02:38:11,880 ARE UNDERNEATH THE NCATS OFFICE 4187 02:38:11,880 --> 02:38:14,920 OF THE DIRECTOR AS OFFICES. 4188 02:38:14,920 --> 02:38:17,560 SO WE KNOW ABOUT OFFICE OF 4189 02:38:17,560 --> 02:38:19,160 STRATEGIC ALLIANCE, THE OFFICE 4190 02:38:19,160 --> 02:38:22,280 OF SPECIAL INITIATIVES, WE HAVE 4191 02:38:22,280 --> 02:38:24,040 OFFICE OF DRUG DEVELOPMENT 4192 02:38:24,040 --> 02:38:25,000 PARTNERSHIP PROGRAMS 4193 02:38:25,000 --> 02:38:26,360 TRANSLATIONAL MEDICINE. 4194 02:38:26,360 --> 02:38:30,000 IN ADDITION THAT YOU HEARD A LOT 4195 02:38:30,000 --> 02:38:32,120 FROM ANNE AND PJ ABOUT RARE 4196 02:38:32,120 --> 02:38:33,480 DISEASE RESEARCH. 4197 02:38:33,480 --> 02:38:35,880 AND THE FOLKS WHO ARE RUNNING 4198 02:38:35,880 --> 02:38:37,120 THIS COUNCIL MEETING IN ADDITION 4199 02:38:37,120 --> 02:38:39,080 TO DOING REVIEW AND GRANTS 4200 02:38:39,080 --> 02:38:40,520 MANAGEMENT IS THE OFFICE OF 4201 02:38:40,520 --> 02:38:42,320 GRANTS MANAGEMENT AND SCIENTIFIC 4202 02:38:42,320 --> 02:38:42,680 REVIEW. 4203 02:38:42,680 --> 02:38:44,120 SO WHAT WE ARE DISCUSSING TODAY 4204 02:38:44,120 --> 02:38:46,160 AND PROPOSING THIS AFTERNOON IS 4205 02:38:46,160 --> 02:38:49,360 ACTUALLY THE ELEVATION OF THESE 4206 02:38:49,360 --> 02:38:52,640 TWO OFFICES, OF AN 4207 02:38:52,640 --> 02:38:53,560 ORGANIZATIONAL LEVEL WITHIN 4208 02:38:53,560 --> 02:38:55,520 NCATS TO HIGHEST ORGANIZATIONAL 4209 02:38:55,520 --> 02:38:57,400 LEVEL WITHIN NCATS AND CREATING 4210 02:38:57,400 --> 02:38:59,160 THE DIVISION OF RARE DISEASES 4211 02:38:59,160 --> 02:39:00,920 RESEARCH INNOVATION OUT OF THE 4212 02:39:00,920 --> 02:39:02,920 OFFICE OF RARE DISEASE RESEARCH 4213 02:39:02,920 --> 02:39:05,400 AND THE DIVISION OF EXTRAMURAL 4214 02:39:05,400 --> 02:39:06,560 ACTIVITIES OUT OF THE OFFICE OF 4215 02:39:06,560 --> 02:39:07,880 GRANTS MANAGEMENT AND SCIENTIFIC 4216 02:39:07,880 --> 02:39:08,240 REVIEW. 4217 02:39:08,240 --> 02:39:12,160 AND WITHIN THOSE OFFICES, OGMS 4218 02:39:12,160 --> 02:39:13,840 OFFICE OF GRANTS MANAGEMENT AND 4219 02:39:13,840 --> 02:39:15,200 SCIENTIFIC REVIEW ALREADY HAVE 4220 02:39:15,200 --> 02:39:17,760 TWO SUE BORT NANT PIECES WHICH 4221 02:39:17,760 --> 02:39:19,080 ARE GRANTS MANAGEMENT AND 4222 02:39:19,080 --> 02:39:20,160 SCIENTIFIC REVIEW PROPOSING 4223 02:39:20,160 --> 02:39:21,960 RENAMING THOSE TO GRANTS 4224 02:39:21,960 --> 02:39:23,200 MANAGEMENT BRANCH AND SCIENTIFIC 4225 02:39:23,200 --> 02:39:25,040 REVIEW BRANCH WITH CREATION OF 4226 02:39:25,040 --> 02:39:27,360 THE OFFICE OF DEA DIRECTOR. 4227 02:39:27,360 --> 02:39:30,280 SO NEXT SLIDE GO INTO MORE 4228 02:39:30,280 --> 02:39:32,360 DETAIL ABOUT WHAT THESE TWO 4229 02:39:32,360 --> 02:39:36,760 CHANGES ENTAIL. 4230 02:39:36,760 --> 02:39:38,280 MAJORITY OF GRANTS ISSUING 4231 02:39:38,280 --> 02:39:39,960 INSTITUTION AND CENTERS AT THE 4232 02:39:39,960 --> 02:39:41,520 NIH HAVE THE GRANTS REVIEW 4233 02:39:41,520 --> 02:39:42,400 FUNCTION MANAGEMENT BY DIVISION 4234 02:39:42,400 --> 02:39:45,360 OF EXTRAMURAL ACTIVITIES. 4235 02:39:45,360 --> 02:39:48,360 WE CURRENTLY HAVE OGSR AS I 4236 02:39:48,360 --> 02:39:49,760 MENTION WE PROPOSE TO ALIGN 4237 02:39:49,760 --> 02:39:51,320 NCATS WITH THE MAJORITY OF THE 4238 02:39:51,320 --> 02:39:53,960 OTHER ICs ACROSS NIH BY 4239 02:39:53,960 --> 02:39:55,400 CREATING THE PROPOSED STRUCTURE 4240 02:39:55,400 --> 02:39:57,840 OF THE DEA DIRECTOR, GRANTS 4241 02:39:57,840 --> 02:39:59,360 MANAGEMENT BRANCH AND SCIENTIFIC 4242 02:39:59,360 --> 02:40:02,120 REVIEW BRANCH, RENAMING THOSE 4243 02:40:02,120 --> 02:40:04,240 AND RENAMING OGMSR TO DIVISION 4244 02:40:04,240 --> 02:40:06,960 OF EXTRAMURAL ACTIVITIES, WHICH 4245 02:40:06,960 --> 02:40:08,880 WOULD BE OVERSEEN COORDINATED BY 4246 02:40:08,880 --> 02:40:10,160 OFFICIAL OFFICE OF DEA DIRECTER 4247 02:40:10,160 --> 02:40:10,560 TO. 4248 02:40:10,560 --> 02:40:11,960 SO THOSE ARE THE CHANGES THAT WE 4249 02:40:11,960 --> 02:40:14,680 ARE PROPOSING TO TAKE PLACE 4250 02:40:14,680 --> 02:40:17,360 WITHIN OGMSR AND ELEVATING 4251 02:40:17,360 --> 02:40:22,400 DIVISION OF EXTRAMURAL 4252 02:40:22,400 --> 02:40:24,600 ACTIVITIES. 4253 02:40:24,600 --> 02:40:26,160 WE PROPOSE TO CHANGE TO DIVISION 4254 02:40:26,160 --> 02:40:27,200 OF RARE DISEASE RESEARCH 4255 02:40:27,200 --> 02:40:30,880 INNOVATION. 4256 02:40:30,880 --> 02:40:33,520 ORDR IS PART OF NCATS SINCE 4257 02:40:33,520 --> 02:40:36,480 CREATION AND YOU HAVE HEARD A 4258 02:40:36,480 --> 02:40:39,120 LOT ABOUT ACTIVITIES THIS 4259 02:40:39,120 --> 02:40:40,240 AFTERNOON SO I DON'T HAVE TO GO 4260 02:40:40,240 --> 02:40:41,920 INTO DETAIL AND TELL YOU HOW 4261 02:40:41,920 --> 02:40:45,800 IMPORTANT ORDR WITHIN NCATS AND 4262 02:40:45,800 --> 02:40:49,120 THE FACT THAT DESPITE RARE 4263 02:40:49,120 --> 02:40:50,120 DISEASE ARE NOT RARE YOU KNOW 4264 02:40:50,120 --> 02:40:50,560 THAT ALREADY. 4265 02:40:50,560 --> 02:40:52,240 WE ARE SEEKING TO ELEVATE OFFICE 4266 02:40:52,240 --> 02:40:53,880 TO DIVISION FURTHER REINFORCE 4267 02:40:53,880 --> 02:40:55,760 IMPORTANCE OF THE WORKS BEING 4268 02:40:55,760 --> 02:40:57,560 DONE AND ALLOW FOR 4269 02:40:57,560 --> 02:40:58,680 SPECIALIZATION FOR CREATION OF 4270 02:40:58,680 --> 02:41:00,640 OPERATING BRANCH WITHIN THIS 4271 02:41:00,640 --> 02:41:01,040 PROPOSED DIVISION. 4272 02:41:01,040 --> 02:41:02,880 THE BRANCHES THAT ARE BEING 4273 02:41:02,880 --> 02:41:03,880 PROPOSED ARE ADVANCE 4274 02:41:03,880 --> 02:41:05,360 THERAPEUTICS AND RESEARCH BRANCH 4275 02:41:05,360 --> 02:41:07,880 OR ATRB AND THAT BRANCH FOCUSES 4276 02:41:07,880 --> 02:41:09,160 ON THERAPEUTICS. 4277 02:41:09,160 --> 02:41:11,560 AND THE SECOND BRANCH WOULD BE 4278 02:41:11,560 --> 02:41:12,880 COLLABORATIVE RESEARCH 4279 02:41:12,880 --> 02:41:15,200 INFORMATICS AND SPECIAL PROGRAMS 4280 02:41:15,200 --> 02:41:17,320 BRANCH WHICH WOULD BE 4281 02:41:17,320 --> 02:41:19,320 ABBREVIATED AND YOU CAN TALK TO 4282 02:41:19,320 --> 02:41:20,640 PJ ABOUT THINKING OF THE 4283 02:41:20,640 --> 02:41:23,280 ABBREVIATION OF THAT, JUST TURNS 4284 02:41:23,280 --> 02:41:25,160 OUT TO SUM THAT WAY BUT FOCUSED 4285 02:41:25,160 --> 02:41:26,920 ON INFORMATICS. 4286 02:41:26,920 --> 02:41:28,520 SO THIS WOULD ENABLE MORE 4287 02:41:28,520 --> 02:41:29,400 TARGETED APPROACH TO STAFFING 4288 02:41:29,400 --> 02:41:30,840 AND OPERATIONS IN THIS PROPOSED 4289 02:41:30,840 --> 02:41:32,560 DIVISION. 4290 02:41:32,560 --> 02:41:35,840 NEXT SLIDE PLEASE. 4291 02:41:35,840 --> 02:41:37,520 I WANT TO TELL YOU ABOUT 4292 02:41:37,520 --> 02:41:38,760 DELEGATION OF AUTHORITY FOR 4293 02:41:38,760 --> 02:41:40,200 APPROVAL ONCE PUBLIC HEARING 4294 02:41:40,200 --> 02:41:42,160 PROCESS IS OVER AND THE TWO 4295 02:41:42,160 --> 02:41:43,360 PUBLIC HEARING SESSION WE WILL 4296 02:41:43,360 --> 02:41:46,880 BE HAVING, AND PROVIDE WE DON'T 4297 02:41:46,880 --> 02:41:49,600 HAVE COMMENTS THAT REALLY SAY WE 4298 02:41:49,600 --> 02:41:51,520 SHOULDN'T BE DOING THIS, THAT IS 4299 02:41:51,520 --> 02:41:54,560 NOT THE CASE. 4300 02:41:54,560 --> 02:41:57,480 THE CHANGE FROM OGMSR TO DEA IS 4301 02:41:57,480 --> 02:41:59,560 DONE BY NIH DEPUTY DIRECTOR FOR 4302 02:41:59,560 --> 02:42:01,000 MANAGEMENT, DR. ALFRED JOHNSON 4303 02:42:01,000 --> 02:42:03,200 REPORTS TO THE ACTING NIH 4304 02:42:03,200 --> 02:42:05,240 DIRECTOR DR. TABAK. 4305 02:42:05,240 --> 02:42:08,240 CHANGE FROM ORDR TO DRI IS MORE 4306 02:42:08,240 --> 02:42:12,400 TRICKY GOING BACK TO NIH BEFORE 4307 02:42:12,400 --> 02:42:15,160 2006 BECAUSE REFORM ACT PUT IN 4308 02:42:15,160 --> 02:42:16,480 LEGISLATIVE LANGUAGE THE NAME 4309 02:42:16,480 --> 02:42:17,440 OFFICE OF RARE DISEASES 4310 02:42:17,440 --> 02:42:19,400 RESEARCH. 4311 02:42:19,400 --> 02:42:21,920 SO ONCE WE HAVE APPROVAL OR WE 4312 02:42:21,920 --> 02:42:23,360 HAVE RECEIVED COMMENTS FROM THE 4313 02:42:23,360 --> 02:42:25,240 PUBLIC HEARING PROCESS, WE WILL 4314 02:42:25,240 --> 02:42:27,960 HAVE TO GO TO THE SECRETARY OF 4315 02:42:27,960 --> 02:42:29,760 HHS TO SEEK APPROVAL TO CHANGE 4316 02:42:29,760 --> 02:42:31,680 THE NAME FROM OFFICE OF RARE 4317 02:42:31,680 --> 02:42:33,440 DISEASE RESEARCH TO DIVISION OF 4318 02:42:33,440 --> 02:42:36,480 RARE DISEASE RESEARCH INVASION. 4319 02:42:36,480 --> 02:42:38,040 THEN FORMULATE LEGISLATION WILL 4320 02:42:38,040 --> 02:42:39,960 HAVE TO BE PASSED FOLLOWING 4321 02:42:39,960 --> 02:42:42,080 APPROVAL FROM SECRETARY BECERRA 4322 02:42:42,080 --> 02:42:43,840 SO THAT WOULD BE THE PROCESS 4323 02:42:43,840 --> 02:42:45,760 FOLLOWING THE PUBLIC COMMENT 4324 02:42:45,760 --> 02:42:46,360 PERIOD. 4325 02:42:46,360 --> 02:42:49,360 NEXT SLIDE PLEASE. 4326 02:42:49,360 --> 02:42:50,480 AS WE TALK ABOUT THE 4327 02:42:50,480 --> 02:42:51,720 OPPORTUNITIES FOR PUBLIC COMMENT 4328 02:42:51,720 --> 02:42:53,720 THIS IS ONE OF THEM. 4329 02:42:53,720 --> 02:42:55,800 THERE WILL BE COUNCIL PUBLIC 4330 02:42:55,800 --> 02:42:57,400 COMMENT OPPORTUNITIES THAT CAN 4331 02:42:57,400 --> 02:42:59,000 BE SAID TO BE EMAIL TO THE EMAIL 4332 02:42:59,000 --> 02:43:03,200 YOU SEE HERE AND NCATS COUNCIL 4333 02:43:03,200 --> 02:43:04,000 INPUT@MAIL.NIH.GOV YOU DON'T 4334 02:43:04,000 --> 02:43:05,600 HAVE TO WRITE THAT DOWN THAT YOU 4335 02:43:05,600 --> 02:43:06,560 HAVE THIS INFORMATION AVAILABLE 4336 02:43:06,560 --> 02:43:07,360 TO YOU. 4337 02:43:07,360 --> 02:43:09,600 IF YOU WISH TO SUBMIT COMMENTS 4338 02:43:09,600 --> 02:43:10,360 AFTER THE PRESENTATION THIS 4339 02:43:10,360 --> 02:43:11,320 AFTERNOON. 4340 02:43:11,320 --> 02:43:13,640 AND YOU HAVE UNTIL FEBRUARY 5 4341 02:43:13,640 --> 02:43:15,800 PUBLIC THIS HEARING IS NOW UNTIL 4342 02:43:15,800 --> 02:43:17,040 FEBRUARY 5 TO SUBMIT PUBLIC 4343 02:43:17,040 --> 02:43:18,240 COMMENT ON THESE RELATED 4344 02:43:18,240 --> 02:43:19,760 CHANGES. 4345 02:43:19,760 --> 02:43:21,920 IN ADDITION WE ARE SEEKING 4346 02:43:21,920 --> 02:43:23,560 PUBLIC COMMENT THROUGH NCATS 4347 02:43:23,560 --> 02:43:25,280 PUBLIC FACING WEBSITE. 4348 02:43:25,280 --> 02:43:27,800 AND WE DO HAVE THE WEB ADDRESS 4349 02:43:27,800 --> 02:43:29,480 HERE AND YOU CAN FIND THAT 4350 02:43:29,480 --> 02:43:32,720 THROUGH NCATS.NIH.GOV. 4351 02:43:32,720 --> 02:43:34,040 THERE IS INFORMATION THERE 4352 02:43:34,040 --> 02:43:34,840 ASSOCIATED WITH THIS THAT YOU 4353 02:43:34,840 --> 02:43:36,600 ARE WELCOME TO LIKE AT AS WELL. 4354 02:43:36,600 --> 02:43:38,760 WE HAVE A PROCESS FOR SUBMITTING 4355 02:43:38,760 --> 02:43:41,360 FEEDBACK THROUGH THE WEBSITE. 4356 02:43:41,360 --> 02:43:43,640 AND THAT FEEDBACK WILL BE 4357 02:43:43,640 --> 02:43:46,360 REVIEWED, WEEK OF JANUARY 21ST, 4358 02:43:46,360 --> 02:43:49,120 AND SUMMARIZE FOR THE RESPECTIVE 4359 02:43:49,120 --> 02:43:50,200 ORGANIZATIONAL CHANGE PACKAGES 4360 02:43:50,200 --> 02:43:51,800 FOLLOWING THAT. 4361 02:43:51,800 --> 02:43:54,200 SO HOPEFULLY I HAVE ONLY TAKEN 4362 02:43:54,200 --> 02:43:55,360 15 MINUTES OF YOUR TIME THIS 4363 02:43:55,360 --> 02:43:57,040 AFTERNOON, I THINK THAT IS MY 4364 02:43:57,040 --> 02:43:58,360 LAST SLIDE, WE TALK ABOUT THE 4365 02:43:58,360 --> 02:44:00,240 PROPOSE REORGANIZATIONAL CHANGES 4366 02:44:00,240 --> 02:44:01,760 AND I WILL CERTAINLY PROVIDE YOU 4367 02:44:01,760 --> 02:44:03,200 WITH THE OPPORTUNITY FOR ANY 4368 02:44:03,200 --> 02:44:05,320 QUESTIONS OR DISCUSSION AS WELL 4369 02:44:05,320 --> 02:44:08,320 SO THANK YOU. 4370 02:44:08,320 --> 02:44:10,480 >> SOUND LIKE A GREAT IDEA. 4371 02:44:10,480 --> 02:44:13,160 >> THANK YOU, KEITH, AND THANK 4372 02:44:13,160 --> 02:44:15,560 YOU, MARSHALL. 4373 02:44:15,560 --> 02:44:18,840 COMMENTS, OTHER COMMENTS? 4374 02:44:18,840 --> 02:44:20,960 >> ALL SOUND LIKE A GOOD IDEA. 4375 02:44:20,960 --> 02:44:22,520 JUST HAVE A QUESTION ABOUT THE 4376 02:44:22,520 --> 02:44:26,360 NAME OF THE DIVISION OF 4377 02:44:26,360 --> 02:44:29,360 (INAUDIBLE) INNOVATION, SO I'M 4378 02:44:29,360 --> 02:44:31,680 WONDERING IF RESEARCH INVASION 4379 02:44:31,680 --> 02:44:33,360 NARROWS A BIT TOO MUCH TO WE 4380 02:44:33,360 --> 02:44:36,400 NEED THE MOD FIE THE RESEARCH. 4381 02:44:36,400 --> 02:44:37,240 -- MODIFY THE RESEARCH. 4382 02:44:37,240 --> 02:44:39,000 THIS IS THE ONLY HOME WE HAVE 4383 02:44:39,000 --> 02:44:41,440 OFFICIALLY AT THE NIH FOR RARE 4384 02:44:41,440 --> 02:44:41,960 DISEASES. 4385 02:44:41,960 --> 02:44:44,920 AND ALL THE INNOVATION AT NIH IS 4386 02:44:44,920 --> 02:44:46,640 REALLY CENTERED AND SO MUCH OF 4387 02:44:46,640 --> 02:44:48,440 WHAT WE HAVE TALKED ABOUT TODAY 4388 02:44:48,440 --> 02:44:52,280 IS NOT JUST ABOUT RESEARCH, 4389 02:44:52,280 --> 02:44:54,160 REALLY ABOUT DATA COLLECTION AND 4390 02:44:54,160 --> 02:44:55,440 DIAGNOSTIC AND NEWBORN SCREENING 4391 02:44:55,440 --> 02:44:56,840 AND OTHER THINGS BUT CERTAINLY 4392 02:44:56,840 --> 02:44:58,760 DO HELP MOVE THE RESEARCH NEEDLE 4393 02:44:58,760 --> 02:45:00,280 TO DO SOME OF THE OTHER THINGS 4394 02:45:00,280 --> 02:45:03,400 FOR THE RARE DISEASE COMMUNITY. 4395 02:45:03,400 --> 02:45:07,920 SO WITHIN THE LARGER CHARGE OF 4396 02:45:07,920 --> 02:45:09,280 NIH AND HEALTH WONDERING P WE 4397 02:45:09,280 --> 02:45:11,480 NEED THE RESEARCH MODIFIER IN 4398 02:45:11,480 --> 02:45:15,040 THE TITLE. 4399 02:45:15,040 --> 02:45:17,040 >> THAT IS GREAT QUESTION. 4400 02:45:17,040 --> 02:45:19,040 WE TALKED ABOUT WHETHER OR NOT 4401 02:45:19,040 --> 02:45:20,720 WE WOULD DIVISION OF RARE 4402 02:45:20,720 --> 02:45:22,840 DISEASE AND DIVISION OF RARE 4403 02:45:22,840 --> 02:45:26,880 DISEASE RESEARCH INVASION. 4404 02:45:26,880 --> 02:45:30,560 IT IS INTERESTING THOSE ARE TWO 4405 02:45:30,560 --> 02:45:32,680 WE WANTED BACK AND FORTH. 4406 02:45:32,680 --> 02:45:33,520 GO AHEAD. 4407 02:45:33,520 --> 02:45:34,640 >> I THOUGHT YOU WERE DONE. 4408 02:45:34,640 --> 02:45:35,200 I'M SORRY. 4409 02:45:35,200 --> 02:45:36,560 I WAS GOING TO SAY WE ARE 4410 02:45:36,560 --> 02:45:40,920 WORRIED ABOUT THE ACRONYM. 4411 02:45:40,920 --> 02:45:45,960 >> YOU ALREADY SAID IT. 4412 02:45:45,960 --> 02:45:48,680 >> I KNOW NIH IS DOING RARE 4413 02:45:48,680 --> 02:45:49,320 DISEASE SO (INAUDIBLE) 4414 02:45:49,320 --> 02:45:53,800 >> WE ARE DOING THE DIRTY WORK. 4415 02:45:53,800 --> 02:45:55,520 CARRYING THE LOAD. 4416 02:45:55,520 --> 02:45:56,960 >> NO, THANK YOU. 4417 02:45:56,960 --> 02:45:58,440 WE -- THAT WAS ONE OF THE 4418 02:45:58,440 --> 02:45:59,560 CONVERSATIONS THAT WE HAVE HAD 4419 02:45:59,560 --> 02:46:01,080 BACK AND FORTH AND WE WELCOME 4420 02:46:01,080 --> 02:46:03,600 ANY OTHER POTENTIAL OTHER 4421 02:46:03,600 --> 02:46:05,160 OPPORTUNITIES THAT WOULD CAPTURE 4422 02:46:05,160 --> 02:46:05,640 THAT NAME. 4423 02:46:05,640 --> 02:46:07,640 I SEE PJ IS ON SCREEN AS WELL SO 4424 02:46:07,640 --> 02:46:09,320 I DON'T KNOW PJ YOU WANT TO ADD 4425 02:46:09,320 --> 02:46:13,400 TO THAT. 4426 02:46:13,400 --> 02:46:17,600 >> NO I MOVED PLACES SO HAPPY 4427 02:46:17,600 --> 02:46:19,160 THE HEAR PEOPLE'S THOUGHTS. 4428 02:46:19,160 --> 02:46:20,440 >> THANK YOU. 4429 02:46:20,440 --> 02:46:22,360 >> YOU CAN GO WITH DIVISION OF 4430 02:46:22,360 --> 02:46:26,280 RARE INNOVATION, DRIVE. 4431 02:46:26,280 --> 02:46:29,160 >> I WOULD HOPE THAT INVASION 4432 02:46:29,160 --> 02:46:30,720 WOULDN'T BE RARE, MARSHALL. 4433 02:46:30,720 --> 02:46:33,760 -- INNOVATION WOULDN'T BE RARE 4434 02:46:33,760 --> 02:46:34,360 MARSHALL. 4435 02:46:34,360 --> 02:46:36,160 >> WE WILL CERTAINLY TAKE THIS 4436 02:46:36,160 --> 02:46:37,200 INTO CONSIDERATION THINKING 4437 02:46:37,200 --> 02:46:38,960 ABOUT WE CAN ALSO MOVE THE EYES 4438 02:46:38,960 --> 02:46:41,520 AND THE Ns AND ALL THAT AROUND 4439 02:46:41,520 --> 02:46:43,200 OR Ds ALL AROUND. 4440 02:46:43,200 --> 02:46:46,560 >> DIVISION OF RARE -- DIVISION 4441 02:46:46,560 --> 02:46:48,680 OF INNOVATION AND RARE DISEASE. 4442 02:46:48,680 --> 02:46:50,960 >> RIGHT. 4443 02:46:50,960 --> 02:46:53,760 >> NOT WHAT YOU INTEND TO SAY. 4444 02:46:53,760 --> 02:46:56,160 YOU ARE -- ELIMINATING RARE 4445 02:46:56,160 --> 02:46:57,920 DISEASE BECAUSE IF YOU PUT RARE 4446 02:46:57,920 --> 02:47:00,440 DEERS RESERGE ININNOVATION AT 4447 02:47:00,440 --> 02:47:01,920 END IT'S NOT CLEAR. 4448 02:47:01,920 --> 02:47:04,080 IT QUOWB INNOVATIONS AT LARGE AS 4449 02:47:04,080 --> 02:47:05,640 OPPOSED TO INNOVATION IN RARE 4450 02:47:05,640 --> 02:47:10,440 DISEASE. 4451 02:47:10,440 --> 02:47:12,480 >> WE ARE AT THE END OF LONG 4452 02:47:12,480 --> 02:47:14,440 WEEK AND ASK US US TO BE 4453 02:47:14,440 --> 02:47:15,640 CREATIVE. 4454 02:47:15,640 --> 02:47:17,200 >> WE FIGURED LATE FRIDAY 4455 02:47:17,200 --> 02:47:18,880 AFTERNOON WE GET THE MOST 4456 02:47:18,880 --> 02:47:19,880 POSSIBILITIES FOR AFTERNOON, 4457 02:47:19,880 --> 02:47:20,360 THAT WAS ANOTHER -- 4458 02:47:20,360 --> 02:47:21,880 >> SINCE I CAME UP WITH THE 4459 02:47:21,880 --> 02:47:23,840 NCATS ACRONYM ORIGINALLY, NOBODY 4460 02:47:23,840 --> 02:47:26,400 WILL GIVE CREDIT FOR ME, WE 4461 02:47:26,400 --> 02:47:31,320 SHOULD LISTEN TO ME. 4462 02:47:31,320 --> 02:47:33,560 >> RAJESH YOU DID A GREAT JOB. 4463 02:47:33,560 --> 02:47:35,360 >> ALL I THOUGHT ABOUT FOR THAT 4464 02:47:35,360 --> 02:47:36,600 PARTICULAR FATEFUL NIGHT WHEN I 4465 02:47:36,600 --> 02:47:40,960 SENT THE EMAIL WAS HERDING CATS 4466 02:47:40,960 --> 02:47:42,160 UNFORTUNATELY. 4467 02:47:42,160 --> 02:47:44,520 >> I THINK AS WE ARE TRYING TO 4468 02:47:44,520 --> 02:47:47,800 GET THE IDEA THAT NCATS IS PLACE 4469 02:47:47,800 --> 02:47:52,000 FOR RARE DISEASE, AT THE NIH AND 4470 02:47:52,000 --> 02:47:53,720 SO I REALLY DO WANT TO GET THAT 4471 02:47:53,720 --> 02:47:56,000 POINT ACROSS, IT IS A HOLISTIC 4472 02:47:56,000 --> 02:47:56,360 IDEA. 4473 02:47:56,360 --> 02:47:59,560 WE DO RESEARCH AT THE NIH OF THE 4474 02:47:59,560 --> 02:48:02,760 COURSE AND SO I THINK HOW WE 4475 02:48:02,760 --> 02:48:04,440 THINK ABOUT MAYBE THE ACRONYMS 4476 02:48:04,440 --> 02:48:06,000 AREN'T AS IMPORTANT OR IF WE 4477 02:48:06,000 --> 02:48:09,080 PICK ONE THAT IS A LITTLE ODD WE 4478 02:48:09,080 --> 02:48:11,760 EMBRACE THAT AND WE USE IT. 4479 02:48:11,760 --> 02:48:13,640 SO I THINK WE HAVE A LOT TO 4480 02:48:13,640 --> 02:48:14,360 CONSIDER. 4481 02:48:14,360 --> 02:48:16,600 WE HOPE FIRST AND FOREMOST WE 4482 02:48:16,600 --> 02:48:17,920 CAN ELEVATE THIS TO A DIVISION 4483 02:48:17,920 --> 02:48:20,040 BECAUSE THAT TO US SEEMS REALLY 4484 02:48:20,040 --> 02:48:21,560 IMPORTANT, REALLY ON PAR WITH 4485 02:48:21,560 --> 02:48:22,600 EVERYTHING ELSE THAT WE ARE 4486 02:48:22,600 --> 02:48:23,320 DOING AT THE NIH. 4487 02:48:23,320 --> 02:48:25,760 SO THAT IS THE GOAL HERE. 4488 02:48:25,760 --> 02:48:30,480 WE WILL GET THE ACRONYM RIGHT. 4489 02:48:30,480 --> 02:48:32,680 I HOPE. 4490 02:48:32,680 --> 02:48:39,880 >> ANY OTHER COMMENTS ON THIS? 4491 02:48:39,880 --> 02:48:42,520 THANK YOU VERY MUCH FOR THAT, 4492 02:48:42,520 --> 02:48:42,960 THANKS. 4493 02:48:42,960 --> 02:48:43,440 >> THANK YOU ALL. 4494 02:48:43,440 --> 02:48:46,160 >> THAT CONCLUDES THE 4495 02:48:46,160 --> 02:48:48,040 PRESENTATIONS FOR TODAY. 4496 02:48:48,040 --> 02:48:50,800 AND THE PRESENTATIONS FOR THE 4497 02:48:50,800 --> 02:48:53,360 29TH NCATS COUNCIL MEETING. 4498 02:48:53,360 --> 02:48:55,120 SO FIRST AND FOREMOST BEFORE WE 4499 02:48:55,120 --> 02:48:58,440 CLOSE OUT I WANT TO OPEN UP TO 4500 02:48:58,440 --> 02:48:59,680 THE FLOOR TO SEE IF THERE ARE 4501 02:48:59,680 --> 02:49:01,440 ANY OTHER ADDITIONAL COMMENTS OR 4502 02:49:01,440 --> 02:49:05,840 QUESTIONS OR IDEAS, AND THEN 4503 02:49:05,840 --> 02:49:07,600 MORE I GUESS TIMELY IS A CHANCE 4504 02:49:07,600 --> 02:49:09,520 TO SAY AGAIN KRISTINA, THANK YOU 4505 02:49:09,520 --> 02:49:12,560 SO MUCH FOR YOUR WORK ON 4506 02:49:12,560 --> 02:49:14,480 COUNCIL, WE ARE GOING TO MISS 4507 02:49:14,480 --> 02:49:16,240 YOU, WE'LL MISS YOUR VOICE BUT I 4508 02:49:16,240 --> 02:49:18,080 DO LOOK FORWARD TO WORKING WITH 4509 02:49:18,080 --> 02:49:20,760 YOU IN YOUR NEW POSITION. 4510 02:49:20,760 --> 02:49:22,520 THANK YOU AGAIN FOR JOINING US 4511 02:49:22,520 --> 02:49:23,080 TODAY. 4512 02:49:23,080 --> 02:49:24,320 I'M SO GLAD YOU WERE ABLE TO 4513 02:49:24,320 --> 02:49:26,840 MAKE IT TODAY AND THANKS FOR 4514 02:49:26,840 --> 02:49:28,200 YOUR EFFORTS ON THIS COUNCIL. 4515 02:49:28,200 --> 02:49:29,920 >> THANK YOU, JONI. 4516 02:49:29,920 --> 02:49:31,160 I APPRECIATE ALL THE WORK YOU 4517 02:49:31,160 --> 02:49:33,920 GUYS ARE DOING AND JUST THE 4518 02:49:33,920 --> 02:49:35,680 CHAMPIONS WE HAVE FOR RARE 4519 02:49:35,680 --> 02:49:39,320 DISEASE NCATS IS INCREDIBLE. 4520 02:49:39,320 --> 02:49:40,760 THRILLED TO HAVE WORKED WITH YOU 4521 02:49:40,760 --> 02:49:42,360 AND KNOW Y'ALL AND LOOK FORWARD 4522 02:49:42,360 --> 02:49:44,040 TO WORKING WITH YOU UNOFFICIALLY 4523 02:49:44,040 --> 02:49:47,840 IN MY CAPACITY AT THE ALLIANCE. 4524 02:49:47,840 --> 02:49:53,560 >> THANK YOU VERY MUCH. 4525 02:49:53,560 --> 02:49:55,400 >> OPEN UP THE FLOOR AND SEE IF 4526 02:49:55,400 --> 02:49:57,760 THERE ARE ANY OTHER QUESTIONS OR 4527 02:49:57,760 --> 02:50:03,080 COMMENTS GOING FORWARD. 4528 02:50:03,080 --> 02:50:03,440 OKAY. 4529 02:50:03,440 --> 02:50:04,960 SOUNDS LIKE YOU ARE READY TO GO, 4530 02:50:04,960 --> 02:50:06,160 HAVE A GOOD WEEKEND. 4531 02:50:06,160 --> 02:50:08,880 I REALLY APPRECIATE AGAIN ALL OF 4532 02:50:08,880 --> 02:50:09,960 YOUR COMMENTS OVER THE LAST 4533 02:50:09,960 --> 02:50:12,400 COUPLE OF DAYS, MY HEAD IS 4534 02:50:12,400 --> 02:50:13,560 SWIMMING, IT WILL BE SWIMMING 4535 02:50:13,560 --> 02:50:14,240 THIS WEEKEND. 4536 02:50:14,240 --> 02:50:16,320 I VERY MUCH APPRECIATE YOUR 4537 02:50:16,320 --> 02:50:18,800 IDEAS AND INPUT ON THE THREE 4538 02:50:18,800 --> 02:50:20,360 GOALS WE WILL CONTINUE TO HONE 4539 02:50:20,360 --> 02:50:20,840 THOSE. 4540 02:50:20,840 --> 02:50:22,840 I FEEL EMPOWERED EVEN MORE SO TO 4541 02:50:22,840 --> 02:50:24,840 DO THAT WITH YOUR ENTHUSIASM AS 4542 02:50:24,840 --> 02:50:25,040 WELL. 4543 02:50:25,040 --> 02:50:27,960 THANK YOU FOR THAT SPECIFICALLY. 4544 02:50:27,960 --> 02:50:29,840 WE LOOK FORWARD TO SEEING YOU IN 4545 02:50:29,840 --> 02:50:30,960 MAY. 4546 02:50:30,960 --> 00:00:00,000 THANK YOU, EVERYONE.