>> WELCOME BACK, EVERYBODY. GREAT TO SEE EVERYBODY. GLAD YOU MADE IT THROUGH THE IMPENDING FLOOD. THEY WERE PLANNING ON DOING ON SITE FILMING FOR NOAH BUT THEY DIDN'T -- NOBODY GOT THE MEMO THAT THE MOVIE HAS ALREADY BEEN COMPLETED SO THEY KEPT THE FLOODING GOING. SO WE HAVE A LOT TO DO TODAY. A LOT OF REALLY IMPORTANT THINGS SO I'M GLAD YOU'RE ALL HERE. WE DID HAVE A FEW PEOPLE FEW COUNCIL MEMBERS AND CAN MEMBERS HAVE PERSONAL OR FAMILY MEDICAL PROBLEMS, I'M AFRAID. SOME QUITE SERIOUS, SOME THAT HAPPENED RECENTLY SO THERE'S SEVERAL PEOPLE WHO ARE MISSING UNFORTUNATELY BECAUSE OF THAT. SO WE'LL SOLDIER ON IN THEIR ABSENCE. THE FIRST ORDER OF BUSINESS IS TO CALL THE SIXTH MEETING OF THE NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES ADVISORY COUNCIL TO ORDER. AND I'LL ASK DR. LEWIS HALL TO CALL -- >> THE 7TH CURES ACCELERATION NETWORK ADVISORY BOARD TO ORDER AS WELL. >> VERY GOOD. SO THE FIRST ORDER OF BUSINESS IS TO DAN WITH MINUTES AND ANY OTHER LOGISTICAL ISSUES. >> GOOD MORNING, EVERYONE. I'M GLAD EVERYONE MADE IT HERE. JUST A COUPLE OF THINGS TO START OUT THE COUNCIL AND CAN REVIEW BOARD MEETING WOULD BE TO REMIND YOU OF THE FUTURE COUNCIL AND CAN REVIEW BOARD SO THE NEXT COUNCIL MEETING IS ON AS INDICATED IN YOUR AGENDA SEPTEMBER 19th SO PUT THAT IN YOUR CALENDARS. THE P CAN REVIEW BOARD WILL HAVE THE VIRTUAL MEETING DECEMBER 12th. STARTING IN 2015 WE ARE ALSO CONSIDERING IF YOU NOTICE A DAY AND A HALF OR STILL A FULL DAY BUT THAT WILL DEPEND ON THE KIND OF AGENDA AND INPUT FROM COUNCIL AND CAN REVIEW BOARD SO STARTING IN JANUARY 2015 IF YOU NOTICE THE MEETING IS GOING TO BE RUNNING ABOUT A DAY AND A HALF OR SO. THE NEXT ITEM IS TO APPROVE THE MINUTES FROM THE LAST COUNCIL MEETING. YOU HAVE COPIES OF THE MINUTES IN FRONT OF YOU AS WELL AS IT WAS MADE AVAILABLE THROUGH ELECTRONIC BOOKS. IF I CAN GET A MOTION TO APPROVE. >> MOVE TO APPROVE. >> ALL IN FAVOR. ALL RIGHT. THANK YOU. >> LET ME TELL YOU HOW WE'RE GOING TO DO THIS. YOU L EEL NOTICE, I WANT TO GO THROUGH THE AGENDA WITH YOU BRIEFLY SO MY DIRECTOR'S REPORT IS SCHEDULED FOR 9 TO 10, IT MAY NOT GO THAT LONG BUT DEPENDS ON ANY QUESTIONS THAT YOU HAVE, IT SHOULD BE SHORTER THAN USUAL. BECAUSE I REALLY WANT TO MAKE SURE WE'RE ON TIME FOR ONE OF THE MAIN EVENTS OF THE MEETING TODAY WHICH IS THE REPORT OF THE NCATS ADVISORY COUNCIL WORKING GROUP AND THE IOM REPORT AND WE HAVE HERE FOR THAT PRESENTATION ALL THREE CO-CHAIRS TWO WHOM ARE ON COUNCIL, SCOTT DISIS AND RON BARTEK IN THE BACK, RON CAN YOU WAVE? RON IS WITH US AS WELL. THEY WILL BE GIVING THAT PRESENTATION. THERE WILL BE PLENTY OF TIME FOR DISCUSSION ABOUT THAT REPORT BECAUSE IT'S AN IMPORTANT MILESTONE FOR NCATS. AFTER LUNCH DR. LEWIS HALL WILL BE GIVING AN UPDATE ON THE CAN REVIEW BOARD PROCESS FOR DECIDING POTENTIAL FUTURE CAN FOCUS AREAS. WE TALKED ABOUT THIS AT THE LAST CAN COUNCIL MEETING. THIS IS A FOLLOW-UP FROM THAT DISCUSSION. WE'LL HAVE REPORTS FROM THE COUNCIL SUBCOMMITTEES ON PATIENT ENGAGEMENT PARTNERSHIPS AND MEDICAL TECHNOLOGIES. THEN WE HAVE A CONCEPT CLEARANCE. SO THERE'S A LOT TO DO TODAY SO WE'RE GOING TO TRY TO STAY ON SCHEDULE. ANY QUESTIONS BEFORE WE GET STARTED? AT LEAST TOO SEE YOUR SMILING FACES. SO IF THERE ARE NO FURTHER QUESTIONS, CAN I START -- THOUGH WE'RE VIDEOCAST, MAKE SURE FOR EVERYBODY BUT SCOTT WEIR, MAKE SURE YOUR HAIR IS COMBED. THOUGH WE'RE VIDEOCAST SCHEDULE CAN I GO EARLY? I CAN. ALL RIGHT. SO I'M GOING TO START WITH THE DIRECTORS REPORT. P >> WHILE CHRIS IS GETTING READY FOR HIS PRESENTATION, WELCOME FRANK WICOLD FROM THE FDA HERE ON BEHALF OF DR. PEG HAMBERG. FRANK DO YOU WANT TO SAY ANYTHING AT THIS POINT? MAYBE NOT AT THIS POINT BUT I WILL CATCH UP WITH YOU IN A FEW MOMENTS. TRAFFIC WAS NOT MY FRIEND THIS MORNING, I APOLOGIZE. WELCOME, FRANK. SO I ALWAYS LIKE TO START OUT WITH REMINDING ALL OF US WHAT OUR MISSION IS TO CATALYZE THE GENERATION OF INNOVATIVE METHODS AND TECHNOLOGIES THAT WILL ENHANCE DEVELOPMENT TESTING AND IMPLEMENTATION OF DIAGNOSTICS AND THERAPEUTICS ACROSS A WIDE RANGE OF DISEASES AND CONDITIONS. TO REMIND YOU THAT WHEN I BECAME DIRECTOR I MADE AN IMPORTANT BUT INFORMAL MODIFICATION, INFORMAL BECAUSE OFFICIALLY CHANGING A MISSION STATEMENT IS A NON-TRIVIAL EXERCISE IN THE FEDERAL GOVERNMENT BUT I THOUGHT THIS WAS IMPORTANT, I HAVE SHOWN YOU THIS BEFORE TO REITERATE DIAGNOSTICS AND THERAPEUTICS ARE IMPORTANT TO ALL OF US BUT DEPENDING HOW YOU THINK ABOUT THIS DEFINITION DEVICES MAY OR MAY NOT BE UNFLUTEDDED IN THIS DEFINITION AND BEHAVIORAL INTERVENTIONS MAY OR MAY NOT B. I WANT TO BE EXPLICIT THAT ALL TYPES OF INTERVENTIONS ARE VERY MUCH WITHIN SCOPE FOR NCATS. THAT'S WHY THIS IS REALLY THE DEFINITION WE USE INTERVENTIONS. BUT INTERVENTIONS NOT SIMPLY INTERVENTIONS, INTERVENTIONS THAT TANGIBLY IMPROVE HUMAN HEALTH THAT OUR JOB IS -- DOES NOT STOP WITH THE DEVELOPMENT OF AN INTERVENTION, WE HAVE TO SHOW IT IMPROVES HEALTH. SO THAT IS ALSO A CHANGE OR MODIFICATION FROM THE ORIGINAL MISSION STATEMENT. AS USUAL, I START WITH SIGNIFICANT CHANGES TO THE NCATS LEADERSHIP AND I'M DELIGHTED TO FINALLY ANNOUNCE OUR HEAD OF THE DIVISION OF CLINICAL INNOVATION, THIS IS PART OF NCATS THAT RUNS THE CTSA PROGRAM. AND SO THIS IS SAN EXTREMELY IMPORTANT -- IS AN EXTREMELY IMPORTANT RECRUIT FOR US, I HAVE BEEN VERY PICKY WITH REGARD TO WHO THIS PERSON WOULD BE BECAUSE IT IS SUCH A CRITICAL POSITION FOR NCATS AND ALL OF NIH. I WOULD ARGUE FOR THE NATION. SO THE WEIGHT WILL HAVE BEEN WORTH IT I THINK. I'M DELIGHTED TO WELCOME PEA TRA KAUFFMAN WHO JOINED US JUST EARLIER THIS MONTH. AS IS OUR PRACTICE, I ASKED HER TO ASSAY A FEW WORDS TO YOU TO INTRODUCE HERSELF TO YOU, TELL YOU ABOUT HER BACKGROUND AND WHAT SHE HOPES TO DO ONCE SHE FINISHES UNPACKING HER BOXES IN HER OFFICE. PETRA, DO YOU WANT TO P COME UP TO THE MICROPHONE? GO TO A MICROPHONE? COME UP HERE BECAUSE THERE ARE PEOPLE ON THE VIDEOCAST THAT WON'T BE ABLE TO HEAR YOU. [APPLAUSE] >> THANK YOU FOR THE KIND INTRODUCTION. I'M ENTHUSIASTIC ABOUT JOINING NCATS AND THE OPPORTUNITY TO HELP STRENGTHEN THE TRANSLATIONAL RESEARCH ENTERPRISE CELEBRATE THE PAST DISCOVERS TO BETTER HEALTH FOR OUR PATIENTS. BEFORE JOINING THE NIH FOUR YEARS AGO I WAS A TENUREDDED FACULTY MEMBER COLUMBIA UNIVERSITY NEW YORK CITY TRAINEDDED IN NEUROLOGY, FROM THAT ACADEMIC TIME I BRING A BROAD SCOPE OF EXPERIENCE RANGING IN E RESEARCH FROM BENCH RESEARCH AND MOLECULAR GENETICS TO CLINICAL RESEARCH, TRIALS AND HEALTH INDs. I THINK ONE EXPERIENCE PARTICULARLY HELPFUL IS IN TEAM SCIENCE BECAUSE I WAS FOCUSING ON RARE DISEASE, I HAD TO WORK WITH MANY OTHER PEOPLE ACROSS THE COUNTRY AND INTERNATIONALLY. I THINK THIS KIND OF EXPERIENCE IN THE SOCIOLOGY AND GOVERNANCE ISSUES WILL BECOME VERY HELPFUL IN LOOKING AT THE CTSA PROGRAM. I HAVE HAD HANDS-ON EXPERIENCE WITH THE CTSA PROGRAM ITSELF MOSTLY ONE CTSA AS CLINICAL VERGER I BENEFITED FROM ACCESS TO RESOURCES AN SUPPORT OF THE CTSA AND I ALSO SERVED ON COMMITTEES FOR RESOURCE ACCESS FOR TRAINING, ACTION POSITION INCLUDING SOME TRANSINSTITUTIONAL IN NEW YORK CITY WHICH IS NOT AN EASY THING. SO I HAVE SOME PERSONAL EXPERIENCE. I MENTION I'M A NEUROLOGIST SO I CURRENTLY SEE PATIENT AT CHILDREN WITH NEUROMUSCULAR DISEASE AND MY PATIENTS HAVE MANY TIMES TOLD ME HOW FRUSTRATE THEY'D ARE WITH THE SLOW PACE OF PROGRESS. THEY WOULD SEE ON THE INTERNET HOW MICE ARE CURED YET IT WILL TAKE MANY, MANY YEARS UNTIL THEY CAN HAVE THE OPPORTUNITY TO ENGAGE IN CLINICAL TRIAL. I SHARED THEIR FRUSTRATION TAKING CARE OF THEM. I FOUND IT OFTEN DAUNTING TO DEAL WITH THE MULTIPLE IRB APPROVALS WITH THE ENDLESS SUBCONTRACTINGND THEN CHALLENGES GETTING PATIENTS ACCESS TO CLINICAL TRIALS. THIS IS IN PART WHAT BROUGHT ME TO THE NIH. THIS OPPORTUNITY TO MAKE A DIFFERENCE. WHEN I CAME TO THE NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE WHERE I HAVE SPENT THE LAST FOUR YEAR OVERSEEING THE EXTRAMURAL CLINICAL RESEARCH PROGRAM I WAS IN A WAY SHOCKED HOW THE EXPERIENCE WAS EVEN, NOT JUST UNIQUE TO MY OWN RESEARCH BUT SO PERVASIVE SO THE MULTI-CENTER TRIALS WE HAD AT THE NINDS HAD TO GO ABROAD TO ENROLL PATIENTS. WHICH IS JUST IMPOSSIBLE TO GET THEM DONE IN A TIMELY MANNER BY JUST RESTRICTING ENROLLMENT TO THE UNITED STATES. ON THE ONE HAND THIS IS WHAT MANY CALL A CRISIS, I THOUGHT ON THE OTHER HAND I WAS GRATIFYING WE WERE ABLE TO MAKE A DIFFERENCE A LITTLE BIT BEHIND THE SCENE AND GET TO SOME STEPS THAT HAVE CHANGED AT LEAST IN NEUROLOGY SOME OF THESE THINGS. AND IN THE INTEREST OF TIME I'LL GIVE YOU A FEW EXAMPLES. ONE EXAMPLE IS THAT WE HAVE CREATED NETWORKS THAT YOU CENTRAL IRB PRE-NEGOTIATED CONTRACTS THAT USE DATA DRIVEN APPROACHES TO IDENTIFY WHERE THE PATIENTS ARE THAT WE WANT TO ENROLL. WE HAVE DONE THIS WITH NEURONECKSIS THAT YOU MAY HAVE HEARD ABOUT FOR EARLY PHASE TRIALS BUT OTHER NETWORKS. THAT HAS REALLY DECREASED THE TIME FROM ABOUT A YEAR IN A TYPICAL CENTER EFFORT TO LESS THAN HALF OF THAT WITH THESE KINDS OF INFRASTRUCTURE IMPROVEMENTS. WE HAVE ALSO BEEN ABLE TO GET SOME REAL SUCCESS EVEN IN VERY RARE DISEASES SUCH AS SPINAL MUSCULAR ATROPHY AND HAVING ENROLLMENT BE ON TARGET. ANOTHER EXAMPLE I WOULD LIKE TO GIVE YOU IS THAT OF HOW WE HAVE CHANGED PATIENT ENGAGEMENT SO WE HAVE TRIED TO HAVE PATIENTS ON BOARD FROM THE BEGINNING ON PROTOCOL WORKING GROUPS AND ALSO IN THE IMPLEMENTATION OVER SIGHT ON DATA AND SAFETY MONITORING BOARDS. WE FOUND THAT IS THE ONLY WAY AND CRITICAL FINDING OUT WHAT MATTERS TO PATIENTS AND FINDING OUT HOW THEY THINK ABOUT THE RISK BENEFIT RATIOS AND ALSO REALLY GETTING THEM ENGAGE AND HELP ENGAGE RESEARCH COMMUNE AT THIS AND ENROLLMENT SO THEY'RE NOT JUST CONSUMERS OF PROTOCOLS OR OF ULTIMATELY WHAT WE WOULD FIND. ANOTHER EXAMPLE I WOULD LIKE TO MENTION IS RELATED TO DATA SHARINGING. SO ONE WAY TO PREPARE DATA SHARING DOWNSTREAM IS TO HAVE DATA COLLECTED IN A MORE STANDARDIZED WAY. I HAD THE PRIVILEGE OF WORKING THIS RON BARTEK AND HIS COMMUNITY CREATING INTERNATIONAL STANDARDS FOR ATAXIA. YOU THINK THAT'S A VERY IMPORTANT FIRST STEP, ESPECIALLY IN RARE DISEASES SO CRITICAL THAT THE DATA CAN SPEAK TO EACH OTHER THE THAT I COLLECTED ACROSS DIFFERENT DISEASES. I HAVE REALLY ENJOYED MY WORK AT THE NIH AND LOVE COMING TO WORK EVERY DAY AND I'M NOW EXCITED ABOUT THE OPPORTUNITY TO COME OVER TO NCATS BECAUSE I CAN BRING THIS BACKGROUND ACADEMIC RESEARCH CTSA EXPERIENCE, BUT ALSO TRACK RECORD AT THE NIH HERE FOR IMPLEMENTING INNOVATION. SO I WANT TO ALSO THANK THE NCATS TEAM AND especially Elaine Collier and the NCATS family making the transition so far making a smooth transition so far tell you how enthusiastic I am about the opportunity to strength tn research enterprise and how I look forward to working with all of you. THANK YOU. [APPLAUSE] SO THE THEMES PETRA WILL NOT SURPRISE YOU WHY I'M ENTHUSIASTIC ABOUT HAVING YOU HERE. THE THREE ISSUES WE TALKED ABOUT INCREASED EFFICIENCY AND EFFECTIVENESS OF CLINICAL STUDIES, PATIENT ENGAGEMENT, AND SHARING OF DATA COMMON DATA ELEMENT, WHAT PETRA DIDN'T TELL YOU, SHE HAS AN A PLUS PLUS REPUTATION AROUND NIH. FOR HER ACUMEN GOOD JUDGMENT TEACH WORK EFFECTIVENESS CHOSEN MULTIPLE TIMES TO BE ON EARLY ON SHE WAS ASKED BY JOCIE BRIGGS AND TOM INSELL TO HELP WITH REVIEW WHAT THE C THETSAs ARE DOING IN CLINICAL RESEARCH RELEVANT TO THE OTHER ICs. SO YOU'LL BE HEARING MUCH MORE FROM PETRA OVER THE NEXT MONTHS AND YEARS AND WE HOPE DECADES. AND CERTAINLY WHAT WE'RE GOING TO BE TALKING ABOUT AFTER THE DIRECTOR'S REPORT ABOUT WORKING GROUP DELIBERATIONS WILL BEAR ON THE WORK WONDERFUL TIME FOR PETRA TO BE STARTING. I WOULD BE REMISS IF I DIDN'T THANK SOMEBODY WHO IS ABSOLUTELY CRITICAL, A BEDROCK TO THIS ORGANIZATION SINCE IT STARTED. SHE MAYBE A SMALL BEDROOM BUT SHE IS A VERY EFFECTIVE FIERCE BEDROCK. AND THAT IS ELAINE COLLIER. ELAINE BECAME IN THE EARLY DAYS OF NCATS DURING JOCIE BRIGGS ACTING DIRECTORSHIP OF THE DIVISION OF CLINICAL INNOVATION HER ACTING CO-DIRECTOR. BECAUSE SHE FOUND THIS PROGRAM IS SO COMPLICATED AND IDIOSYNCRATIC THAT SHE NEEDED A CO-PILOT FOR THE WORK SHE WAS DOING. AFTER I TOOK OVER I WAS HEAD OF DCI FOR THREE MONTHS SO I COULD LEARN THE PROGRAM AND QUICKLY REVERTED TO AN EVEN MORE MODEL WHICH PUT MORE RESPONSIBILITY ON ELAINE'S SHOULDERS TO MAKE HER ACTING DIRECTOR OF DCI WHICH SHE HAS DONE FOR THE LAST 14 MONTHS I THINK. ELAINE AND I TRAVELED TO ABOUT 25 CTSAs TOGETHER, SO I ALWAYS FEEL LIKE WE SHOULD HAVE T-SHIRTS WITH CTSA WORLD TOUR 2013. I FOUND HER A CONSISTENT AND VERY WISE COUNCIL ON SCIENTIFIC AND ORGANIZATIONAL ISSUES. SHE KNOWS THE THIS PROGRAM LIKE THE BACK OF HER HAND, SEEMS TO KNOW EVERYTHING PROFESSIONAL AND PERSONAL ABOUT EVERY ONE OF THE P PIs SO IF YOU'RE A PI OF THE PROGRAM OR LISTENING OUT THERE, ELAINE TAKES A DEEP INTEREST IN ALL OF YOU PROFESSIONALLY AND PERSONALLY. I REALLY WANT TO THANK ELAINE FROM THE BOTTOM OF MY HEART ON THE PART OF NOT ONLY MYSELF BUT CENTER AND SUCCESS WE HAVE AND PETRA HAS WILL BE BUILT ON THAT FOUNDATION. THANK YOU AGAIN. [APPLAUSE] SO THE OTHER GOOD NEWS IS THAT ELAINE IS NOT GOING ANYWHERE. SHE'S NOW BECOMING WHAT SHE WAS ACTUALLY BEFORE SHE GOT INTO HER CURRENT POSITION WHICH IS SENIOR ADVISER TO THE DIRECTOR. I HAVE A WHOLE LONG LIST OF THINGS THAT I NOW WANT HER TO FIX NOW THAT SHE'S DONE WITH THE CTSA SHE CAN WORK ON GOING TO THE LONG LIST OF TO DOs THAT WE HAVE. SO YOU'LL BE HEARING MORE FROM HER. THE NCATS ORGANIZATION, TO UPDATE YOU IN ADDITION TO PETRA, HERE IS WHAT THE ORG CHART LOOKS LIKE, YOU HAVE SEEN THIS BEFORE, I SHOW THIS TO YOU EVERY COUNCIL MEETING. THE OBJECTIVE HERE IS TO HAVE THE RED WRITING GO AWAY, THE RED WRITING OF THE ACTING DIRECTORS. WHEN WE STARTED ESSENTIALLY ALL THESE BOXES WERE RID. MANY ARE FILLED IN NOW. THE CURRENT STATUS HERE IS OF COURSE PE THETRA HAS JOINED AS DIRECTOR OF THE DCI THAT STARTED MAY 4TH. THE SCIENTIFIC DIRECTOR SEARCH COMMITTEE PROCESS IS WELL ALONG. THEY ARE DUE TO GIVE ME THE SHORT LIST FOR THIS POSITION BY THE END OF THE MONTH. I HOPE TO BE ABLE TO ANNOUNCE A PERMANENT SCIENTIFIC DIRECTOR AS OF THE SUMMERTIME. THE OFFICE OF RARE DISEASES WAS ONE OF THE ORGANIZATION THAT HAD A PERMANENT HEAD, STEVE GROFT, WHEN WE STARTED BUT OF COURSE AS YOU KNOW HE RETIRED BACK STEVE RETIRED AND IS BACK A COUPLE OF DAY AS WEEK BUT RETIRED OFFICIALLY IN FEBRUARY SO THOSE RECRUITMENT PLANS ARE IN PROGRESS AND I SHOULD REMIND YOU PAMELA KENNIS DEPUTY DIRECTOR IS ACTING DIRECTOR OF ORDR. THE OGMSR POSITION WE PLAN TO READVERTISE FOR. PAMELA, WHEN PAMELA STARTED THE THING THAT I ASKED HER TO FOCUS ON MOST INTENTLY WAS THIS POSITION THIS IS NCATS DOES THINGS, WANTS TO DO THINGS DIFFERENTLY WE NEED A HEAD OF OGMSR WHICH WHO IS GOING TO BE ATYPICAL. SOMEBODY WHO INNOVATES IN THIS SPACE. WE CONSIDER INNOVATION AND SCIENTIFIC REVIEW OF GRANTS MANAGEMENT, AND WORKING WITH YOU, THE COUNCIL AND CAN BOARD WE WANT TO DO DIFFERENTLY SO WE WANT SOMEBODY TO FOCUS ON INNOVATION IN THIS SPACE. PREVIOUS SEARCH COMMITTEE BEFORE MY TIME SO WE READVERTISED THAT POSITION AND PAMELA IS HAHN CO-ING THAT EFFORT. SO AT LEAST WE HOPE BY THE TIME WE MEET THESE SHOULD BE FILLED AND WELL ALONG TO FILLING. I TOLD THE FOLKS WHEN THEY START NOBODY CAN RETIRE UNTIL THE YEAR 2200. SO NO MORE RED ON THIS DIAGRAM ONCE PEOPLE START. THESE POSITIONS ARE LIKE A ROACH MOTEL. YOU CAN MOVE IN BUT YOU CAN'T MOVE OUT. THE FY 14 APPROPRIATION HAD NON-NOT BEEN PASSED THE LAST TIME WE MET, IT WAS PASSED TWO DAYS AFTER THE LAST COUNCIL MEETING. IN CASE YOU ARE INTO THIS STUFF PUBLIC LAW 113-76. IF DO YOU WANT LOOK THIS UP. THE NIH APPROPRIATION WAS JUST A BIT SHY OF $30 BILLION. THE NCATS APPROPRIATION WAS $633 MILLION. THIS WAS AN INCREASE OVER THE PREVIOUS YEAR, HALF OF WHICH WAS DESIGNATED FOR -- IN THE APPROPRIATION FOR THE CTSA PROGRAM. THE OTHER APPARENT INCREASE ACTUALLY WASN'T AN INCREASE AT ALL, IT WAS AN ACCOUNTING CHANGE, MULTIPLE PROGRAMS AS YOU PROBABLY REMEMBER PART OF DCI AND OTHER PROGRAMS, WERE IN THE COMMON FUND SO THEY NEEDED TO BE MOVEDDED OUT OF THE COMMON FUND AND INTO THE NIH BASE THIS IS THE NEW THERAPEUTIC USES PROGRAM, THE BRIDGES PROGRAM AND THREE MOLECULAR LIBRARIES PROGRAMS, ALL THOSE WERE MOVED OUT OF THE COMMON FUND INTO OUR APPROPRIATION. THAT DIDN'T GIVE AN INCREASE, ACTUALLY GAVE A SMALL DECREASE IN OUR BUDGET FOR A VARIETY OF ACCOUNTING REASONS. BUT AN APPARENT INCREASE FOR ACCOUNTING CHANGE TO PUT MONEY INTO THE BASE FOR THE FIRST TIME, GOOD NEWS FOR US BECAUSE IT MADE US HOLE AS A CENTER. THAT WE HAD APPROPRIATED PROJECTS THE PROGRAMS ASSIGNED TO US WHEN NCATS WAS FOUND. SO THIS IS ANOTHER -- IF YOU WANT TO THINK OF IT, THIS IS KIND OF A BIRTH DEFECT OF THE NCATS THAT THESE MONEYS WERE NOT IN OUR APPROPRIATION WHEN WE STARTED AND IT CAUSED SIGNIFICANT PROBLEMS FOR US NOT TO HAVE CONTROL SO ON THE FY 15 APPROPRIATION STATUS YOU REMEMBER FY 15 STARTS OCTOBER 1st OF THIS YEAR SO NOT FAR OFF. SO OF COURSE WE HAD BEEN WORKING ON THIS APPROPRIATION FOR SOME TIME, IT WAS RELEASED LATE, AROUND THE TIME OF THE STATE OF THE UNION ADDRESS, RELEASED LATE THIS YEAR, IT'S A PUBLIC DOCUMENT AND YOU CAN REACH A SUMMARY OF THIS AND ONLY THE ORIGINAL LANGUAGE ON OUR WEBSITE AND YOU'LL HAVE THESE SLIDES SO YOU CAN GO LOOK IN DETAIL ABOUT WHAT THE BUDGET JUSTIFICATION SAYS. OF COURSE THERE'S A LOT OF UNCERTAINTY ABOUT THE LONGEVITY OF ANY SUCH CONGRESSIONAL JOB JUSTIFICATION GIVEN WHAT'S GOING ON IN THE CONGRESS AT THE MOMENT BUT THIS IS AN IMPORTANT AT LEAST POLICY DOCUMENT THAT LAYS OUT WHAT THE ADMINISTRATION FEELS IS THEIR PRIORITIES FOR THE NEXT FISCAL YEAR SIMILARLY IT LAYS OUT WHAT NIH FEELS ARE ITS PRIORITIES FOR THE NEXT FISCAL YEAR AND WILL COME BACK TO THIS A LITTLE BIT IN THE CANDICE CUSHION BECAUSE IN OUR WE REQUESTED NIH REQUESTED AN INCREASE IN THE CAN BUDGET WHICH THEN WOULD GIVE THIS GROUP ABILITY TO DO SOME OF THE THINGS THAT WE HAVE BEEN TALKING ABOUT AND HOPED TO DO. SO STAY TUNEDDED HOW THAT HAPPENS. SO THERE WERE TWO CONGRESSIONAL APPROPRIATIONS HEARINGS THIS YEAR, ONE IN THE HOUSE, ONE IN THE SENATE, I WAS PRIVILEGED TO TAKE PART THE ONE IN THE SENATE WAS ASKED TO CONTRIBUTE TO ATTEND THIS ONE. IN THE HOUSE HEARING THERE WEREN'T ANY DIRECTION -- ANY QUESTIONS DIRECTLY ABOUT NCATS BUT FRANCIS AND OTHERS MENTIONED THE TISSUE CHIP PROGRAM AND SICKLE CELL PROGRAM THAT IS GOING ON IN THE TREND PROGRAM PART OF DCI. IN THE SENATE HEARING FRANCIS THROUGH ME A NICE SOFTBALL WHEN THERE WAS A QUESTION ABOUT A PRIMATE RESEARCH AS FRANCIS IS WANT TO DO HE TURNED THIS INTO A QUESTION ABOUT ANIMAL ALTERNATIVES AND TURNED TO ME AND SAID HEY, TALK TISSUE CHIP. SO I SEIZE THE MOMENT AND HERE I AM HOLDING UP A KIDNEY CHIP AT THAT HEARING. AND IT WAS A BIG HIT. WE HAD HEARING WITH SENATE FOR DURBIN AND OTHER CONGRESSIONAL MEMBERS A FEW MONTHS BEFORE. I WAS ABLE TO WOW HIM WITH A NUMBER OF TISSUE CHIPS AS WELL, GAVE HIM ONE SO THIS PROGRAM THAT DAN AND KRISTEN RUN IS EXTREMELY HELPFUL FOR SHOW AND TELL PURPOSES. THERE IS NOTHING LIKE SHOWING A CONGRESSMAN AN ACTUAL DEVICE AND I KNOW PAUL KNOWS THIS, IT'S IT'S IMPORTANT HOW A MOLECULE INTERACTS WITH ITS MOLECULAR TARGET AND HAS SIGNAL TRANSDUCTION. SOMEHOW THAT SPEAKS TO HIM NOT SURE WHY THAT IS BUT IF YOU SHOW HIM A KIDNEY AND CAN SHOW HIM THIS IS WHERE THE BLOOD COMES IN, THIS IS WHEN THE URINE GOES OUT, YOU CAN SAY WOW, I KNOW WHAT URINE IS AND WHAT BLOOD IS. I'M A SCIENTIST. IT'S REALLY IMPORTANT FOR US BECAUSE IT REALLY -- WE ARE A TRANSLATIONAL CENTER. SO WE THINK VERY INTENTLY ABOUT TRANSLATING WHAT WE DO, NOT ONLY MEDICALLY BUT VERBALLY. SO THIS KIND OF THING IS VERY IMPORTANT. POLICY UPDATES, A VERY IMPORTANT MILESTONE FOR US THE REPORT. IT IS IN YOUR PACKET SENT APRIL ST.. IT'S REQUIRED BY CONGRESS IN THE APPROPRIATIONS ACT THAT CREATED US IT IS QUITE A WONDERFUL DOCUMENT THAT HIGHLIGHTS OUR MISSION MILESTONES PROGRAMS, INITIATIVES P AND WE PLAN TO USE THIS AS A COMMUNICATION DOCUMENT TO REALLY EXPLAIN WHAT WE'RE ALL ABOUT. I HAVE TOLD YOU BEFORE THAT IN MY CONVERSATIONS AND I'LL GET TO THIS IN A LITTLE BIT ABOUT HOW MUCH OUTREACH I DO AND THE REST OF THE STAFF DOES, I HAVE NOT INFREQUENTLY BEEN ASKED OKAY YOU ARE THE NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES. SO YOU GUYS DO LIKE LINGUISTICS, RIGHT? LISTENING WESTIC, TRANSLATIONAL SCIENCES. YOU STUDY -- ISN'T THAT A FUNNY THING FOR NIH TO DO? CONGRESSMEN HAVE ASKED ME THIS. I HAD A SCIENTIST WHO ASKED ME ONCE, YOU'RE AN ENTIRE CENTER AT NIH THAT FOCUSES HOW RNA IS MADE INTO PROTEIN. ISN'T THAT SPECIFIC? THIS TRANSLATIONAL SCIENCE. SO WE ARE CONSTANTLY THINKING ABOUT WAYS THAT WE CAN MAKE -- DEFINE WHAT WE DO IN MUCH MORE UNDERSTANDABLE TERMS, IT'S SOMETHING WE TALKED ABOUT HERE BEFORE AND I KNOW YOU'RE HELPING US WITH. IT IS A UNIQUE BURDEN THAT WE HAVE THE OTHER INSTITUTES DON'T HAVE. THEY DON'T HAVE TO EXPLAIN WHAT THEY'RE SOLVING WHAT CANCER IS BEFORE THEY EXPLAIN TO PEOPLE WHAT THE CANCER INSTITUTE IS DOING FOR INSTANCE. COMMUNICATION AND POLICY STAFF SPENT ENORMOUS AMOUNT OF OF TIME ON THIS AND I HOPE YOU ENJOY READING IT. THE PICTURES ARE REAL PICTURES FROM NCATS PROGRAMS, NOT STOCK PHOTOS SO ENJOY THAT AS WELL. SO THERE IS AN INTEREST PROGRESS SYCES GOING ON IN CONGRESS NOW, CALLED 20th CENTURY CURES ROUND TABLE, CURE THERE'S A ROUND TABLE ABOUT IT, A VERY INTERESTING PROCESS T OUTCOME OF WHICH IS NOT YET CLEAR BUT CERTAINLY IT IS A VERY POSITIVE DEVELOPMENT SO REPRESENTATIVE FRED UPTON CHAIR OF HOUSE CONGRESS COMMITTEE AND DEANNA DEJET, ANOTHER MEMBER OF THE COMMITTEE HAVE BEEN PUSHING THIS PROCESS OFFING IN EXPANSIVE WAYS HOW THE CONGRESS MIGHT HELP NOT ONLY THE NIH BUT OTHER SCIENCE AGENCIES WITH INNOVATION. AND THERE WAS A VERY INTERESTING ROUND TABLE ON 20th CENTURY CURE WITNESS WHICH THE TISSUE CHIP PROGRAM WAS MENTIONED. I'M GOING CALL THOUGH SHE DOESN'T KNOW I'M GOING TO DO THIS, SWALLOW THAT COFFEE SO YOU CAN SAY SOMETHING ABOUT THIS. MARGARET ANDERSON WAS ON THAT PANEL AND SOME OF YOU MAY HAVE SEEN IT'S BEEN WEBCAST AND IT'S ON THE WEB BUT MARGARET, CAN YOU SAY A FEW WORDS ABOUT YOUR IMPRESSIONS HOW THAT WENT AND WHERE IT MIGHT BE GOING? >> IT'S CLEARLY A LONG TERM PROCESS MR. UPTON IS ENGAGING IN, AS YOU SAID NOT SURE ANYONE KNOWS EXACTLY WHAT THE OUTPUT IS, WHAT I THINK IS IMPORTANT TO NOTE PARTICULARLY ABOUT THIS ROUND TABLE WAS THE DIVERSITY OF PARTICIPATION FROM THE MEMBERS OF CONGRESS AND THE STRENGTH OF THEIR PARTICIPATION. THEY WERE THERE THE ENTIRE TIME, THEY WERE ENGAGED. I THINK IT WAS A MUCH MORE CONDUCIVE SETTING TO INFORMATION SHARING AND LEARNING THAN PERHAPS TRADITIONAL HEARING WHERE IT'S JUST KIND OF SERIAL PRESENTATION. THERE WAS MORE BACK AND FORTH AND -- SO ANY TIME THAT YOU HAVE MEMBERS OF CONGRESS AND COMMITTEES THAT ARE THAT ENGAGED IN THE FUTURE OF INNOVATION I THINK IS A POSITIVE THING. THERE IS A LOT OF DIFFERENT ACTIVITY ON THE HILL RIGHT NOW AS YOU REFERENCED, I THINK IT'S IMPORTANT EVERYBODY IS LIENENING TRYING TO FIGURE OUT HOW TO HELP STRUCTURE PARTICIPATION AND INPUT FOR AD GOOD OUTCOME. >> SO STAY TUNED. THOSE STUDENTS OF HISTORY WILL REMEMBER THE WORD CURES HAS BEEN USED BEFORE AND THE AMERICAN CURES ACT, THE CURES ACCELERATION NETWORK CAME FROM ONE OF THESE EFFORTS BEFORE SMUGLY THE P CAST REPORT FITS INTO THIS PATTERN. ALL OF OUR HOPE IS THIS WILL REALLY RESULT IN SOMETHING SUBSTANTIAL. IT WAS INTERESTING FOR ME TO LISTEN TO THIS WEBCAST BECAUSE AT TIMES I FELT LIKE I WAS LISTENING TO A DISCUSSION OF NCATS MISSION. IT REALLY WAS VERY FOCUSED ON TRANSLATION AND WAYS TO DO THIS BETTER. SO YOU'RE NOT SUPPOSED TO READ ALL THIS, THIS IS IS A SHOCK AND AWE SLIDE THAT IS SUPPOSED TO INDICATE HOW LITTLE I HAVE BEEN IN THE OFF FIT FOR THE LAST THREE MONTHS. SO I SPENT A LOT OF TIME ON THE ROAD BOTH TALKING TO PEOPLE ABOUT WHAT NCATS DOES AND HAVING THEM TELL ME WHAT THEY THINK PRIORITIES OUGHT TO BE. THIS IS ACROSS ACADEMIA, NOT ONLY WITHIN MAJOR GROUPS LIKE AAMC BUT I SPENDS A LOT OF TIME AT INDIVIDUAL ACADEMIC DEPARTMENTS AND VISITING7 – CTSAs. I WAS ACTUALLY AT MOUNT SINAI, THIS WASN'T ON HERE, I WAS AT MOUNT SINAI ON WEDNESDAY AND I WAS IN MADISON UNIVERSITY OF WISCONSIN MADISON ON MONDAY. VISITING THEIR CTSAs, WITH FOUNDATION, OTHER PARTS OF THE GOVERNMENT, AND INDUSTRY AS WELL. I THINK AS I MENTIONED THIS BEFORE, THE TENOR OF THESE VISITS HAS CHANGED RADDEDLY IN THE LAST YEAR AND A HALF SINCE I BECAME DIRECTOR. I'LL USE THE TERM MARCH GREAT GROWTH USES THAT IS LEANING IN. WHEN I VISIT THESE PLACES, THERE IS INTENSE INTEREST IN WHAT NCATS IS DOING, WE AND YOU ARE VIEWED AS IN MANY CASES THE LEADERS OF THE INTERESTING STUFF AS PEOPLE SAID WHAT'S GOING ON AT NIH. IT'S GRATIFYING TO HEAR THAT BECAUSE IT'S TRUE. CERTAINLY OUR AMBITIONS ARE MUCH GREATER THAN WE HAVE BEEN ABLE TO DO SO FAR. THE LAST YEAR AND A HALF IN MY MIND HAS -- I HAVE OFTEN DESCRIBED THIS IS BUILDING A FOUNDATION, MUCH HAS BEEN UNDERGROUND UNVISIBLE BECAUSE IF YOU LOOK ACROSS YOU CAN'T SEE A DEEP STRONG FOUNDATION BUILT. I FEEL LIKE NOW WE'RE AT THAT POINT. I THINK WE'RE AT AN INFLECTION POINT WE'RE ABLE TO DO SOME VERY IMPORTANT TRANSFORMATIONAL THINGS. WHAT I HEAR WHEN I TRAVEL IS THAT EVERY P COMPONENT OF THE RESEARCH INFRASTRUCTURE IS YEARNING FORS TO DO THAT AND LOOKING FORS TO DO THAT. PART OF THIS CTSA SITES I SHOW YOU THIS, MAP OF THE CTSAs, THEY'RE IN BLUE. THE PLACES I VISITED MOSTLY WITH ELAINE BUT SOME OF THEM BY MYSELF ARE IN RED. AND THE PLACES THAT ARE SCHEDULEDDED TO BE UPCOMING ARE IN GREEN. EVERY ONE THAT I VISIT I LEARN SOMETHING ABOUT TRANSLATIONAL SCIENCE, PROBLEMS WE MIGHT HELP SOLVE, AND I HOPE THEY LEARN SOMETHING ABOUT IDEAS THAT WE HAVE TO IMPROVE THIS PROCESS AS WELL. SO OUR COMMUNICATIONS HAS CONTINUED TO RAMP UP. WE HAVE INCREASINGLY -- INCREASING PRESENCE ON SOCIAL MEDIA AS WELL AS CONVENTIONAL MEDIA. WE ARE ENGAGED IN A VERY IMPORTANT TOTAL WEBSITE REDESIGN AT THIS POINT. THE WEBSITE IS NOT BAD AS IT IS BUT IN ALL OF OUR VIEWS DOES NOT REFLECT THE INNOVATION THAT WE WANT TO PORTRAY. THIS WEBSITE WAS PUT TOGETHER VERY QUICKLY IN THREE MONTHS THE EARLY DAYS OF NCATS SO IT IS -- IT WAS A RUSH J HAS A BIT OF A LOOK OF A RUSH JOB, HELD TOGETHER WITH SCOTCH TAPE AND CHEWING GUM. COMMUNICATION STAFF HAS DONE AN AMAZING JOB IN DOING THIS. P WE'RE COMPLETELY REDESIGNING IT TO BE MORE USER FRIENDLY AND FOCUSED ON THE INNOVATION THAT'S SO CENTRAL TO WHAT WE DO. ALSO TO INCLUDE A LOT OF EDUCATIONAL MATERIALS AND INFORMATION ABOUT THE PROJECTS WE DO AND HOW TO WORK WITH US. NEWS LETTERS YOU KNOW YOU GET THIS, I KNOW Y'ALL READ IT COVER TO COVER, CAN YOU READ THAT COVER TO COVER? WHAT'S THE ENTREPRENEUR BITE TO BITE? I DON'T KNOW WHAT THAT IS. I HAVE PAD TO iPAD FRONT TO BATCH NUMBER OF DIRECTORS MESSAGE, I HAVE A MESSAGE THAT COMES OUT EVERY MONTH OR TWO, WE AIM EVERY MONTH, WE PRETTY MUCH KEEP ON EVERY MONTH I SHOULD SAY. IN MARCH IT WAS ON A TECHNOLOGY I'LL TOUCH ON IN A SECOND, NEW TECHNOLOGY FOR IDENTIFYING COMBINATION TREATMENT, IN APRIL WAS ABOUT 3-D DISEASE MODELS NOT JUST TISSUE CHIP PROGRAM BUT TWO OTHERS, THERE'S AN UPCOMING MESSAGE WE HOPE TO BE RELEASED ON MONDAY THAT INCLUDE COMMENTS ABOUT ALL OF YOU. MAYBE EVEN PICTURES OF YOU. SO THESE ARE FEATURE ARTICLES THAT ARE THERE. FEATURE ARTICLES ON THE WEBSITE ONE ON THE COMBINATION SCREENING PLATFORM BUT THERE'S ANOTHER VERY INTERESTING ARTICLE ON THE PROGRESS A VARIETY OF IRB CTSA INSTITUTIONS HAVE MADE ON VARIOUS MODELS OF IRB RELIANCE HARVARD AND OHIO AND CALIFORNIA AND ELSEWHERE. AND PAM DAVIS WHO COULDN'T BE WITH US TODAY BECAUSE HER HUSBAND HAD MAJOR SURGERY LAST WEEK UNEXPECTEDLY, DOING FINE BUT HER MODEL IN HOUGH OH IS ONE OF THOSE -- OHIO IS ONE THAT WAS FEATURED IN THIS ARTICLE. NCATS HAS PRESS, THESE WERE STORIES ABOUT US OR TALK ABOUT OUR PROGRAMS OR THINGS THAT WE DO. IT WAS A VERY NICE ARTICLE IN THE TRADE PRESS ABOUT LILY PORTIA, HEAD H OF STRATEGIC ALLIANCES. IN RARE DISEASE DAY, WHICH TO REMIND YOU COMES THE LAST DAY OF FEBRUARY EVERY YEAR, IT'S ON LEAP YEAR, IT IS ON FEBRUARY 29th, IT'S A RARE EVENT. ON -- THE OTHER DAY IS ON FEBRUARY 28th. THIS WAS AN UNUSUAL YEAR NOT ONLY BECAUSE STEVE GROFT WAS FEATURED IN HIS RETIREMENT BUT LEONARD LANCE WHO IS A VERY COMMITTED PUB CAN CONGRESSMAN FROM NEW JERSEY COMMITTED TO RARE DISEASE CAME IN QUITE INSIDESIVE TALK ABOUT RARE DISEASES, FRANCIS COLLINS GAVE A NICE TALK, I GAVE A TALK WHICH I'M NOT SURE IS NICE OR NOT. LEAVE IT UP TO YOU. THEN JOHN GALLON WHO RUN IT IS THE CLINICAL CENTER SPOKE, BILL CHEN FROM PHARMA, AND AMY MARCUS WHO IS A PULL LESSER PRIZE WINNER AT THE WALL STREET JOURNAL, WHO DID THE MULTI-PART STORY THAT I HOPE A LOT OF YOU HAVE SEEN. AND SEVERAL HUNDRED PEOPLE ATTENDED THAT. I JUST WANT TO THANK DAVID ECSTEIN FROM ORDR ON THAT FROM THE CLINICAL CENTER. THIS IS -- JUST SOME PICTURES, THIS IS STEVE GROFT, I PRESENTED THIS PLAQUE THAT I PRESENTED TO HIM AT THE LAST COUNCIL MEETING. I PRESENTED TO HIM THREE OTHER TIMES AT THREE OTHER RETIREMENT EVENTS. WE GOT TO LET HIM KEEP IT THE LAST TIME. I KEPT GIVING IT TO HIM AND TAKING ATE IT BACK. THIS IS LEONARD LANCE WE RECOLLECT WENT ON A TOUR THE CLINICAL CENTER BEFORE. THIS IS AMY AMY MARCH DOWN HERE. THERE WAS A NICE PIECE WITH STEVE USDON, I DON'T KNOW HOW MANY GET UP SUNDAY MORNINGS AND WATCH THESE. THESE THE BIOMEDICAL EQUIVALENT OF MEET THE PRESS. SO THIS IS SUNDAY MORNING TALK SHOW. AND THERE WERE VERY NICE PIECE, CAMEOS BY FREDA AND TONY FAUCI TAKEN FROM THE TRANSLATIONAL VIDEO Y'ALL TOOK PART IN. YOU CAN WATCH THE ENTIRE 30 MINUTES IF YOU HAVEN'T AFTER THE DIRECTOR'S REPORT YOU CAN WATCH ME THERE AS WELL. I THINK WE'RE GOING TO SHOW YOU A CLIP. SO LET JEFF SPENCER DO THIS. >> BIOVINT THIS WEEK. >> IN DECEMBER 2011 NIH OPENED THE DOORS TO A NEW CENTER NCATS, THE NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES. NCATS MISSION TO RE-ENGINEER THE WAY BASIC RESEARCH IS TRANSLATED INTO MEDICINE. SUPPORTERS SAY NCATS WILL SOLVE PROBLEMS, ACADEMICS AND INDUSTRY CAN'T TACKLE ON THEIR OWN BUT SKEPTICS INSIDE ACADEMIA AND INDUSTRY AND EVEN AT NIH SAY IT DIVERTS NIH CORE MISTAKING ON TASKS THAT SHOULD BE LEFT TO DRUG COMPANIES. THIS WEEK WE'LL TRY TO SEPARATE THE HOPE FOR THE HYPE WITH THE FIRST NCATS DIRECTOR DR. CHRIS AUSTIN. >> PLEASED TO BE JOINED WITH DR. CHRIS AUSTIN. CHRIS, WHAT'S TRANSLATION AND WHY DOES NIH NEED A CENTER TO ADVANCE IT? >> TRANSLATION IS THE PROCESS BY WHICH AN OBSERVATION IN THE LABORATORY OR IN THE CLINIC IS TRANSFORMED INTO AN INTERVENTION, A DRUG, A DIAGNOSTIC A DEVICE, WHICH IS SHOWN TO IMPROVE HUMAN HEALTH, THERE ARE 7,000 DISEASES THAT AFFECT THE HUMAN FAMILY. IT WILL TAKE OVER A THOUSAND YEARS FOR EVERY DISEASE TO BE TREATABLE. I WOULD ARGUE THAT THAT IS SIMPLY NOT AN ACCEPTABLE ANSWER. >> I WONDER AGAIN WHY IS IT SOMETHING NIH NEEDS TO A DRUG COMPANY HAS A FANTASTIC INCENTIVE TO GET THESE DRUGS TO GET NEW DRUGS USED AS WIDELY AS POSSIBLE, AS QUICKLY AS POSSIBLE. AND THEY PUT FANTASTIC RESOURCES INTO DOING THAT. SO IT MAYBE QUESTIONABLE BUT THEY DO IT. WHY DOES NIH? >> BECAUSE WHEN NCATS WAS FOCUSED ON PARTICULARLY ARE THE GENERAL PRINCIPLES BY WHICH WE CAN DO EACH OF THESE THINGS BETTER. GIVE YOU AN EXAMPLE. IT'S WELL KNOWN THAT MOST PATIENTS WHO ARE PRESCRIBED A MEDICINE NEVER FILL A PRESCRIPTION OR THEY ONLY TAKE IT ONE MONTH AND THEY STOP. THE BEST DRUG IN THE WORLD IS NO GOOD FOR YOUR HEALTH IF YOU DON'T TAKE THE MEDICINE. THIS IS PHYSICIAN BEHAVIOR, PRESCRIBING BEHAVIOR, PATIENT BEHAVIOR, AND THIS IS AN ISSUE OF IMPLEMENTATION SCIENCE, QUITE DIFFERENT FROM THE FUNDAMENTAL PRE-CLINICAL WORK NCATS DOES. >> WHAT ARE YOU DOING? IT'S A CHALLENGING PART. >> THROUGH THE CTSA PROGRAM, THE CLINICAL SINCE PROGRAM, A WONDERFUL NETWORK OF 62 ACADEMICCAL CENTERS ALL OVER THE COUNTRY, NIH'S BIGGEST SINGLE PROGRAM, WE HAVE REALLY PUSHED THE IDEA OF PATIENT ENGAGEMENT AND COMMUNITY ENGAGEMENT FROM THE VERY BEGINNING OF PROJECTS. WHAT ARE THE REASONS THAT PATIENTS DO NOT TAKE THE MEDICINES THAT MIGHT BE PRESCRIBED THEM, IF YOU DON'T FEEL THEY'RE PARTNERS WITH THE PRESCRIBERS, IN UNDERSTANDING WHAT THESE MEDICINES CAN DO FOR THEM. , IT'S AN INTERESTING DEVELOPMENT YOU PROBABLY TALKED ABOUT IT ON THE SHOW THAT PATIENT GROUPS ARE BECOMING MUCH, MUCH MORE ACTIVE THAN THE DEVELOPMENT AND UTILIZATION OF MEDICINES FOR THEIR DISEASES. WE THINK THIS IS A TRANSDEVELOPMENT AND ACTUALLY CHALLENGED NCATS TO HAVE IN OUR PEOPLE AND GRANTEES AND INTERNAL SCIENTISTS TO INVOLVE PATIENTS IN EVERY PROJECT WE DO FROM THE VERY BEGINNING. >> CAN YOU GIVE OTHER EXAMPLES OF THE WAY THAT YOU TRIED TO CATALYZE CHANGE? >> WE HAVE A WONDERFUL COLLABORATION CALLED NEW THERAPEUTIC USES WITH 8 PHARMACEUTICAL COMPANIES, A GREAT EXAMPLE OF ADDRESSING A SYSTEMATIC PROBLEM IN COLLABORATION WITH THE PRIVATE SECTOR CHARACTERISTIC OF EVERYTHING WE DO. SO THE PROBLEM IS BECAUSE OF THE DRUG DEVELOPMENT DRUG APPROVED FOR HUMAN USE BY THE FDA, THERE ARE ABOUT TEN WHICH HAVE BEEN IN PEOPLE AND FAIL. OFTEN FOR EFFICACY REASONS OR BUSINESS REASONS. SO WE TEAMED UP WITH THESE COMPANIES TO SAY GOSH, A LOT OF WORK GOING INTO THESE DRUGS, HOW ABOUT WE GO TO THE ACADEMIC COMMUNITY P AND SAY LOOK, WHAT IDEAS DO YOU HAVE FOR OTHER DISEASES THAT THESE DRUGS MIGHT BE USED FOR? AND HAVE REPURPOSED THOSE, SO CALLED, IN ACTUALLY NINE DIFFERENT DISEASES NOW, THAT ARE IN PATIENTS RIGHT NOW IN COLLABORATION BETWEEN NCATS AND ACADEMIC ORGANIZATIONS AND THE PHARMACEUTICAL COMPANIES WHO MADE THE DRUGS AND HAVE ALL THE DATA ON THEM. >> AND WHAT ABOUT THAT, YOU SEE WHETHER THE NINE DISEASES IS GOING TO WORK IN THEM? >> GOOD QUESTION. CLINICAL TRIALS IN GENERAL TAKE ABOUT A YEAR BEFORE YOU GET FIRST INDICATION SO I WOULD SAY ABOUT A YEAR. SOME ARE GETTING ANIMAL STUFF SOME, A LITTLE EARLIER THAN THAT. BUT I SHOULD SAY THIS WHOLE PROGRAM WAS ABOUT $13 MILLION, VERY SMALL AMOUNT OF MONEY THAT COULD CATALYZE NINE NEW DRUGS. SOMETIMES THIS IS LIKE A FOOTBALL GAME, YOU CAN IMAGINE THE DRUGS ON THE 2-YARD LINE AND THEY NEED ONE QUICK PLAY TO GET THEM OVER THE GOAL LINE AND HELP A LOT OF PATIENTS. THAT'S THE KIND OF THING THAT NCATS DOES. >> I WANT TO EMPHASIZE THESE THINGS ARE POSSIBLE. RAPID ADVANCEMENTS IN TRANSLATION ARE POSSIBLE. THEY WEREN'T POSSIBLE WHEN I WAS IN TRAINING 40 YEARS AGO. THEY ARE POSSIBLE NOW, IT'S A MATTER OF WILL, AND HAVING THE SCIENCE AND OPERATIONAL SYSTEMS TO DO IT. >> BIOCENTURY THIS WEEK. >> IN DECEMBER 2011 NIH OPENED THE DOORS TO A NEW CENTER, NCATS. >> WE DON'T HAVE TO WATCH IT AGAIN. JEFF HAS IT AS A LOOP ON HIS -- THIS IS JEFF SPENCER BY THE WAY, WE HAVE BEEN TOGETHER A LONG TIME IN COMMUNICATIONS. ONE OF THE MODEL SYSTEMS WE'RE INTERESTED IN IS THIS ONE, THERE'S 3-D MODEL SYSTEMS FOR TESTING SAFETY AND EFFICACY OF NOVEL THERAPEUTICS LAST TIME I TOLD YOU ABOUT A NOVEL COLLABORATION WITH DE NOVO AND SAN DIEGO ON PRINTING RETINAS AND NOW PRINTING SKIN. THIS IS A PARTNERSHIP WITHIN SPHEREO LOOKING AT POTENTIAL OF THE SYSTEM OF 3 DIMENSIONAL MODELS OF CANCER, PANCREATIC OVARIAN CANCER AS BETTER MODELS FOR TESTENING CANCER DRUGS. COUPLE OF SPECIFIC EXAMPLES, BECAUSE I WANT TO INCREASINGLY NOW DIG DOWN AND REDUCE THE PRACTICE FOR YOU, SOME OF THE EFFECTS THAT -- SOME OF THE SUCCESSES THAT NCATS HAS HAD IN A VERY DOWN TO EARTH KIND OF WAY. SO THIS IS THE FIRST PAPER THAT WAS PUBLISHED BY A PUBLIC PRIVATE TEAM. IN THIS CASE BETWEEN NCATS, PHARMACEUTICALS, A COMPANY IN BOSTON AND NOVARTIS ON A REALLY ACCELERATED PROGRAM TO DEVELOP INHIBITORS OF MUTANT DEHYDROGENASE WHICH SOME OF YOU PROBABLY KNOW HAS BEEN SHOWN TO BE MUTATED IN MANY TYPES OF CANCER. THIS IS A GREAT EXAMPLE OF NOT ONLY PRODUCING PROOF OF PRINCIPLE COMPOUNDS BUT ALSO PUTTING ALL THE DATA IN THE PUBLIC DOMAIN SO OTHERS CAN USE THESE DATA. ANOTHER COLLABORATION WITH LARGE PHARMA IS PART OF THE TREND PROGRAM, IT'S AN INTERESTING REPURPOSINGING PROGRAM, THIS IS A PARATHIGH RA HORMONE RECEPTOR MODULATOR DEVELOPED BY LILY THEY HOPE FOR HOST OWE PROSIS, THEY CAME TO US AS PART OF THE TREND PROGRAM, THERE'S A LOT OF WORK BOTH EF DA SANE TOX WORK AND CLINICAL WORK THAT HAS TO BE DONE TO BE ABLE TO USE THIS FOR IDIOPATHIC HYPOTHYROIDISM, WE WOULD NEVER DO IT ON OUR OWN BUT CAN WE WORK WITH YOU AND DO THIS SO IT'S A GRATEFUL EXAMPLE OF A VARIATION ON THE NEW THERAPEUTIC USES THEME THAT YOU HEARD ABOUT. THE REASON THIS IS SO IMPORTANT FOR US NOT ONLY AS SCIENTIFIC MEDICAL POINT OF VIEW BUT GIVE LIFE TO THE IDEA THAT SOMEHOW NCATS AND PHARMA ARE COMPETING WHICH THEY NEVER HAVE. BUT THIS WAS A CANARD IN THE COMMUNITY FOR WHILE AND THIS PROJECT REALLY PUTS THAT TO BED. SO THE IOM REPORT YOU WILL HEAR ABOUT, I WANT TO REMIND YOU THIS CAME OUT LAST JUNE ABOUT 11 MONTHS AGO NOW, A LITTLE LESS THAN THAT. HAS THESE SEVEN RECOMMENDATIONS, YOU WILL HEAR MORE ABOUT THIS JUST AFTER THIS PRESENTATION. FROM THE WORKING GROUP, SO THIS GROUP HAS BEEN EXTREMELY BUSY SINCE THE LAST COUNCIL MEETING MY SPECIAL ASSISTANT CHRISTINE CATIL AROUND HERE SOMEWHERE, RAISE YOUR ARMS SO HERB KNOWS WHO YOU ARE. HAS DONE THAT WORK AS A SHERRY NICHOLS WHO HELPED WITH THIS. THEY HAVE HAD A REMARKABLE GROUP OF PEOPLE WHO HELPED ON THIS ADVISORY COUNCIL WORKING GROUP THAT YOU WILL BE HEARING ABOUT. SO STAY TUNED. ANOTHER THING WE HAVE DONE WHICH IS REALLY IN THE CTSA ABOUT THE CTSA PROGRAM, WITHIN THAT PROGRAM SINCE THE LAST COUNCIL MEETING, WE HAD DONE THIS AT THE TIME OF THE COUNCIL MEETING BUT IT WAS JUST GETTING GOING. WAS TO IMPLEMENT THIS, IF I GO BACK TO THIS FIRST STRENGTH AND LEADERSHIP OF THE CTSA PROGRAM BY NCATS AND NUMBER TWO RECONFIGURING THE GOVERNANCE, THIS WAS ONE OF THE FIRST THINGS WE DID TO IMPLEMENT THIS RECOMMENDATION, THAT WE ABOLISHED THE PREVIOUS STEERING COMMITTEE WHICH HAD 90 PEOPLE ON IT. ANYBODY WHO IS TRYING TO DRIVE A BUZZ DRIVEN BY 90 -- BUS KNOWS IT'S HARD TO GET TO IT GO ANYWHERE IF YOU HAVE 90 PEOPLE STEERING IT SO WE REPLACED THAT STEERING COMMITTEE AND EXECUTIVE COMMITTEE OVER THAT WITH A NEW GROUP WHICH IS AN APPROPRIATE SIZE TO ACTUALLY BE ABLE TO HAVE SOME IMPACT ON THE PROGRAM. THIS IS CHAIRED BY THE HEAD OF DCI, WAS LYNN COLLIER, NOW PETRA, CO-CHAIRED BY NORA DISIS WHICH IS OUR -- THE NCATS SWISS ARMY KNIFE. SHE DOES VIRTUALLY EVERYTHING THAT WE ASK HER TO DO. AND A NUMBER OF REALLY GREAT PEOPLE PIs, 12 PIs AND A NUMBER OF PEOPLE FROM NCATS WHO ARE MOVING FORWARD, A COUPLE OF THINGS, YOU WILL HEAR MORE ABOUT THIS, FIRST THINGS WE DID WHEN WE PUT THE STEERING COMMITTEE TOGETHER IS WE ASK THEM TO CONSIDER TWO THINGS, ONE IS WHAT TO DO WITH ALL OF THE APPROXIMATELY 200 COMMITTEES THAT HAD GROWN UP IN THE UNITED NATIONS BUREAUCRACY WHICH I THINK EVERYBODY AGREED PRETTY MUCH AND THIS IOM TALKED ABOUT THIS, RESULTS IN A TYRANNY OF COMMITTEE MEETINGS WITH UNCLEAR OUTCOMES IN MOST CASES. THEY VOTED TO ABOLISH THOSE COMMITTEES, CLEAR CUT AND START FROM THE BEGINNING. THIS RESULTED IN SOME PAIN. WHEN YOU DO THAT BECAUSE THERE WERE COMMITTEES AMONG THE 200 BUT THEIR OPINION I AGREED WITH THIS, THAT IT WAS BEST TO CLEAR CUT, AND LIKE IN CLEAR CUTTING YOU'RE NOT JUST DOING IT FOR CLEAR CUTTING YOU WANT NEW YOUNG TREES TO GROW UP IN THEIR PLACE. STORE THE PHOENIX, WHATEVER ANALOGY BURNOOSE THE GROUND AND GROWS BACK UP AGAIN. SO NEW COMMITTEES WHICH ARE PROBLEM FOCUSED, JUST GETTING STARTED AGAIN. THE OTHER THING WE ASKED THEM TO DO IS FOCUS ON A FEW CONSOR SHALL, A FEW PRIORITIES THAT WOULD BE CONCOURSE YUM PRIORITIES FOR THE CTSA, MORE THAN ONE CTSA AND FOCUSED ON SOME IMPORTANT CENTRAL ISSUES IN EFFICIENCY OF THE CLINICAL PROCESS AND THEY CHOSE TWO. ONE HARMONIZING ELECTRONIC MEDICAL RECORDS FOR PATIENT RECRUITMENT AND FURTHER HARMONIZING ACROSS THE NETWORK IRB SYSTEM. THESE PROJECTS MOVING RAPIDLY NOW UNDER THE GUIDANCE OF MEMBERS OF THE STEERING COMMITTEE AND P PIs IN THE CTSA PROGRAM, AND THESE ARE VERY EXCITING DEVELOPMENTS, THEY ARE WHAT WE ALL HOPEDDED FOR AND I'M COULD NOT BE MORE EXCITED ABOUT WHAT THE STEERING COMMITTEE AND THE CTSA PIs AT LARGE AS A WHOLE ARE DOING IN THESE PROGRAMS. SO STAY TUNED, A LOT IS GOING TO BE HAPPENING NOW. >> VERY EXCITING THING THAT HAPPENED LAST WEEK, THERE'S SOMETHING CALLED THE HHS IGNITE AWARD. THIS IS AN AWARD THAT'S GIVEN EVERY YEAR TO A A TEAM WITHIN THE DEPARTMENT OF HEALTH AND HUMAN SERVICES FOR SOMETHING WHICH IGNITES FUNDAMENTAL CHANGE WITHIN THE ORGANIZATION. THIS IS A PROGRAM THAT -- AWARD THAT WAS GIVEN TO INFORMATICS TEAM PRE-CLINICAL INNOVATION AND INFORMATICS FOLKS AND THE COMMISSIONER AT FDA TO DO SOMETHING WHICH FREDA WOULD SAY, I REMEMBER HE SAYING THAT A COUPLE OF YEARS AGO. THE BIG ISSUE WITH NCATS, THERE'S REALLY IMPORTANT THINGS TO DO BUT THEY'RE NOT SEXY. I ASSIGNED FREDA TO BE THE CHIEF SEXY OFFICER. SO TRYING TO MAKE WHAT WE DO SEXY. AMONG THE OTHER RESPONSIBILITIES, THIS IS A CLASSIC PROBLEM. SO THE PROBLEM HERE IS FDA AND OTHER REGULATORY AGENCIES DO NOT HAVE A SUBSTANCE REGISTRATION SYSTEM THAT ALLOWS THEM TO FIGURE OUT WHAT THE SUBSTANCE ARE, SUBSTANCES ARE THAT THEY'RE REGULATING WHICH YOU MIGHT THINK THAT'S JUST INSANE. HOW COULD THAT BE. IT IS NOBODY'S FAULT, IT'S HISTORICAL ARTIFACT OF COMPUTER SYSTEMS CHANGING SO RAPIDLY, IT'S A BIT LIKE THE FAA STILL USING VERY OLD TRACKING SYSTEMS. SO OUR NCATS INFORMATICS FOLKS WHO HAVE A VERY DEEP LONG STANDING RELATIONSHIPS WITH THEIR COLLEAGUES OUR COLLEAGUES AT THE FDA RECOGNIZE THIS INTERNALLY AND STARTED WORKING ON IT A YEAR AND A HALF AGO. FDA IS FUNDING NCATS IN PART TO DO THIS PROJECT. BUT IT IS A JOINT PROJECT. WE DEVELOPED A ARCHIVE SUBTRANSREGISTRATION INFORMATION NOT ONLY WITHIN FDA BUT GLOBALLY. OF COURSE REGISTRATIONS NOW ARE NOT JUST DONE IN ONE COUNTRY BUT GLOBALLY NOW. COMPANIES DON'T TO DEAL WITH THE VARIOUS SYSTEMS AND NCATS ESTABLISHED THAT. THIS IS A REALLY IMPORTANT AWARD. A REALLY -- THIS IS WHAT WEIR DOING WITH ROCKEFELLAR WHO IS PI OF CTSA ROCKEFELLAR BUT THIS PROJECT IS COMPLETELY INDEPENDENT OF THE THE CTSA PROGRAM. THAT'S A COMPOUND CALLED ROCK 2 WHICH STANDS OR FOR ROCKEFELLAR UNIVERSITY COMPOUND 2, THAT WE HELPED -- WE DID MA DISNAL COMMENT ON AND PUBLISHED IN SCIENCE TRANSLATIONAL MEDICINE A COUPLE L OF YEARS AGO, PLATELET INTEGRIN ANTAGONISTS SAME CLASS AS THE ANTAGONIST ON THE MARKET BUT IF YOU'RE CARDIOLOGISTS IT CAUSES A CONFIRMATIONAL CHANGE WHICH CAUSES AUTOANTIBODIES AGAINST THE BODY AND CAUSES THROMBO CYTOPENIA. THESE DON'T CAUSE THE AUTOANTIBODIES DON'T CAUSE THE THROMBO CYTOPENIA. SO WE'RE WORKING WITH THE FDA TO ESTABLISH IND AND PFIZER HAS INTEREST IN THIS BUT WE'LL PROBABLY HAVE TO TAKE IT THROUGH INITIAL CLINICAL TRIALS FIRST. ANOTHER REALLY GROUND BREAKING DRUG ABILITY ISSUE WHICH NCATS SUCCEEDED IN DOING, WHOLE CHASE OF UBIQUITIN SPECIFIC PROTEASES WHICH ARE INVOLVED IN THE A VARIETY OF RESPONSES, THIS HAPPENS TO BE INVOLVED IN DNA DAMAGE RESPONSE. AND MANY, MANY PEOPLE LOOK FOR INHIBITS TO OF THESE ENZYMES, PLEIOTROPIC THERAPEUTIC INDICATIONS BUT NO ONE WAS ABLE TO DO THIS AND OUR GROUP WORKING WITH INVESTIGATORS AND EXPERT AT UNIVERSITY OF DELAWARE HAS MANAGED TO DO THIS AND THIS IS PUBLISHED IN NATURE CHEMICAL BIOLOGY. AND THE STRATEGIC ALLIANCE GROUP IS SEEKING TO ADVANCE THE TECHNOLOGY. SO YOU MIGHT ASK YOURSELF WHY DID THIS HAPPEN? IT HAPPENED IN THE SAME PRINCIPLE THAT YOU HEAR AT NCATS OVER AND OVER AND OVER AGAIN, INVESTIGATOR UNIVERSITY OF DELAWARE KNOWS MORE THAN ANYBODY ABOUT DUBS. OUR PEOPLE DON'T KNOW HOW TO SPELL UBIQUITIN BUT HE DOESN'T KNOW HOW TO SPELL DRUG. WHEN THE GROUPS COME TOGETHER THAT'S WHEN THE MAGIC HAPPENS. THIS IS A VERY EXCITE TECHNOLOGY, THERE'S AN ISSUE YEARS AND YEARS HOW TO DEVELOP, IDENTIFY COMBINATION OF DRUGS THAT MIGHT BE USEFUL PARTICULARLY IN CANCER AND INFECTIOUS DISEASE BUT IN DISEASES IN GENERAL. BEEN A VERY DIFFICULT PROBLEM, IT'S REALLY BEEN A TECHNICAL PROBLEM, A COMPOUND MANAGEMENT PROBLEM, INFORMATICS PROBLEM, COMMONTORICS THAT TRIED THIS A NUMBER OF YEARS AGO AND WENT OUT OF BUSINESS TRYING TO DO IT BECAUSE OF TECHNICAL CHALLENGES. OUR MULTI-DISCIPLINARY TEAM IS NOW SOLVED THIS PROBLEM. NOT ONLY PROMULGATED PROOF OF PRINCIPLE HERE, REMEMBER 3-D DEVELOP DEMONSTRATE DESISNATE. IT'S AN ENGINEERING COMPOUND MANAGEMENT INFORMATICS SOLUTION PARTICULARLY USING ACOUSTIC DISPENSING. THEY DEMONSTRATED UTILITY WITH LOU INSTITUTE STATE, INVESTIGATOR AT THE CLINICAL CENTER AND THE TECHNOLOGY IS DISSEMINATED. ALL THE TECHNIQUES, ALL THE PROTOCOLS, ALL THE INFORMATIC ANALYSIS SOFTWARE IS ON OUR WEBSITE. ANOTHER THING WEIR INTO IS PATIENT INITIATED SCIENCE, WE HAVE DONE MANY PROJECTS WITH PATIENT FOUNDATIONS AND FOUND THEM -- FOUND THOSE PROJECTS MOVE FORWARD AT A FASTER RATE THAN OTHER PROJECTS BECAUSE THE INVOLVEMENT OF PATIENTS. JUST RECENTLY WE STARTED TWO NEW PROJECT, ONE WITH THE ALPHA 1 FOUNDATION, ALPHA ONE TRYPSIN FOUNDATION, THE ALPHA 1 PROJECT. AND ONE WITH THE THAN'S HOPE FUND WORKING ON A NEUROLOGICAL DISORDER CALLED GIANT EOME NEUROPATHY. ONE THING ABOUT NUMBER TWO, I THINK A LOT OF YOU KNOW ALPHA 1 FOLK AND THEY ESTABLISH EXCELLENT PEOPLE. ON THE HANNA'S HOPE STORY IS INTERESTING. WHEN I MET THE MOM ABOUT A MONTH AFTER HER DAUGHTER WAS DIAGNOSED, SHE CAME TO ME WITH A FOLD THEIR1"l– LOOKED LIKE SOMETHING THAT ONE OF MY DATES WOULD HAVE MADE IN THIRD GRADE, ABOUT LEVEL OF HER KNOWLEDGE AT THE TIME BUT SHE WAS SO COMMITTED AND SO OBVIOUSLY FULL OF NATIVE INTEL CONSENSUS WE DECIDED TO WORK WITH HER TO DEVELOP THIS PROJECT. SHE HAS DEVELOPED THIS PROGRAM INTO A SOPHISTICATED SCIENTIFIC PROGRAM AND RAISED MONEY TO SPONSOR A FELLOW IN OUR LAB WORKING ON ASSAY DEVELOPMENT TO SCREEN FOR COMPOUNDS WHICH WILL BE ABLE TO TREAT THIS DISORDER. IT'S A GREAT EXAMPLE OF MOVING FORWARD IN A PROGRAM AND NCATS CATALYZING ADVANCES THAT OTHERWISE WOULD NOT EVER HAPPEN, AT LEAST NOT ON THIS TIME SCALE. THE TISSUE CHIP PROGRAM, NO UPDATE ON A LOT OF SCIENCE HERE, YOU'LL BE HEARING ABOUT THE REVIEW PROCESS FOR GOING TO SECOND PHASE OF THE PROJECT LATER TODAY FROM THE SO CALLED UH-2 TO 3. BUT I JUST WANT TO POINT OUT, THIS PROGRAM IS GETTING A LOT OF PRESS IN THE SCIENTIFIC, NOT JUST CONGRESSIONAL HEARINGS, SO YOU WILL SEE THE JOURNALS WHERE PAPERS PUBLISH ON THIS, THERE IS AN INTERESTING ARTICLE NATURE MEDICINE ABOUT ONE OF THESE RESEARCHERS MODELING CARDIOMYOPATHY IN PATIENTS WITH BAR SYNDROME, A RARE DISEASE, ONE INTERESTING THING YOU MIGHT WANT TO ASK DAN ABOUT LATER IF YOU CAN GET HIM TO GET OUT OF HIS EXPECT SEC MODE FOR THE COUNCIL AND INTO HIS RUNNING THE TISSUE CHIP PROGRAM MODE, IS THAT THOUGH THE PROGRAM STARTED AND STILL IS FOCUSED ON P DEVELOPING SYSTEMS TO TEST SAFETY AND EFFICACY OF NOVEL THERAPEUTICS, TURNING OUT A LOT OF EARLY APPLICATIONS OF THESE PROGRAMS ARE TURNING OUT TO BE USEFUL FOR DISEASE MODELING. PERHAPS THOSE WILL BE THE FIRST INDICATION. BECAUSE WHAT WE TALKED ABOUT SHOW AND TELL IS TRUE, WE WANTED TO HAVE YOU BE ABLE TO EXPERIENCE SHOW AND TELL T. SO WE HAVE THIS MODULE FOR YOU, SO I'M GOING TO PASS THIS AROUND, THANK KRISTEN WHO IS WORKING WITH DAN ON THIS. THIS BELIEVE IT OR NOT, THIS IS A BRAIN SO YOU CAN PUT THIS RIGHT HERE I SUPPOSE. OR ACTUALLY MORE SPECIFICALLY A BLOOD BRAIN BARRIER. I WILL PASS THIS AROUND BUT YOU MIGHT HAVE HEARD THAT ONE OF THE ISSUES WITH THIS PROGRAM HAS BEEN THAT THOUGH THE CHIPS THEMSELVES ARE VERY SMALL, MICROFLUIDICS AND PUMPS AND THINGS REQUIREDDED TO FEED EACH OF THESE, HAS BEEN ENORMOUS UNTIL NOW. BUT THIS ACTUALLY IS THE ENTIRE UNIT, THESE ARE THE PUMPS THAT PUMP IN THE SOLUTIONS. AND THIS IS THE CHIP HERE. SO LET ME TELL YOU WHAT YOU'RE GOING TO LOOK AND I'M GOING TO PASS IT AROUND, IT'S A BLOOD BRAIN BARRIER CHIP. THIS IS PSEUDO COLORED HERE BUT THE RED IS ARTIFICIAL BLOOD. THE BLUE IS TISSUE CULTURE MEDIA FOR THE NEURAL CELLS AND GLIA AND BLOOD VESSEL CELLS WHICH ARE ON THE OTHER SIDE, THE NEURONS AND GLIA ON THE OTHER SIDE, AND AS A MEMBRANE BETWEEN THAT ACTS AS A BLOOD BRAIN BARRIER. SO WHAT YOU'LL SEE IS THE RED GOES IN AS THE BLOOD ARTIFICIAL BLOOD, THE BLUE CSF IF YOU WANT TO THINK THAT WAY, AND THEN THIS GOES IN AND COMES OUT INTO THE PURPLE HERE, THE PURPLE IS WHEN THE RED AND BLUE GO TOGETHER. YOU CAN INFUSE COMPOUNDS AND SEE WHETHER THEY PASS THE BLOOD BRAIN BARRIER FROM THE RED TO THE BLUE. SO I WILL WILL GIVE THIS TO THE ETHICIST FIRST BECAUSE HE WILL INTERPRET THIS. SO THERE WILL BE A QUIZ AT THE END SO YOU MAY STUDY THAT, AS WE GO ON. THERE'S A COUPLE OF TOYS UP HERE THAT YOU SHOULD LOOK ATLANTA THE BREAK. WHERE IS KRISTEN? HERE SHE IS. KRISTEN KNOWS MORE ABOUT THIS THAN ANYBODY. DAN HER BOSS ON -- IF YOU PUSH HIM HE MIGHT BE ABLE TO TELL YOU SOMETHING TOO. THE NEW THERAPEUTIC USES PROGRAM, THIS IS THE PROGRAM KRISTIN COLVIS RUNS, THERE'S EXCITING THINGS GOING ON BUT FOR YOU AND THE FOLKS IN CYBER SPACE AND THE NEW FUNDING OPPORTUNITY ANNOUNCEMENTS ARE RELEASED FOR THE NEW GROUP OF COMPOUNDS, THERE'S A ASSISTANCE WEBINAR ON.* THIS, THIS IS ROUND TWO FOR THE NEW THERAPEUTIC USES PROGRAM AND THE PRE-APPLICATIONS ARE DUE IN JULY SO THERE'S A COUPLE OF NEW THING, I DON'T KNOW IF -- RIGHT. A COUPLE OF THINGS HERE, NEW COMPOUNDS BUT COMPOUNDS AVAILABLE FOR PEDIATRIC INDICATIONS AND ADDITIONAL FUNDING THAT'S AVAILABLE FOR DOING THE TOX WHICH IS REQUIRED FOR PEDIATRIC INDICATIONS, THAT WAS NOT POSSIBLE THE LAST TIME. EXTRA CELLULAR RNA IS ANOTHER PROGRAM OFFICE OF STRATEGIC INITIATIVES ESPECIAL INITIATIVES AND DAN TAGLY RUNS THIS PROGRAM AS WELL. THERE'S A WHOLE BUNCH OF PROGRAMS WITHIN THAT -- WITHIN THAT PROGRAM FOCUSING ON DATA MANAGEMENT BIOGENESIS OF EXTRA CELLULAR RNA, USE OF THERAPEUTICS, BIOMARKERS AND PROFILES OF THESE EXTRA CELLULAR RNAs AND VARIOUS TISSUES AN DISEASE STATES. THERE IS A REALLY INTERESTING RAPIDSLY GROWING AREA OF BIOLOGY AND SO THIS -- GROUP HAS PUT TOGETHER AN EXTRA CELLULAR RNA PORTAL THAT HAS PROJECTS PUBLICATIONS, ET CETERA SO IF YOU'RE INTERESTENING UNDERSTANDING MORE AB EXTRA CELLULAR RNA AS BIOMARKERS, AS POTENTIAL THERAPEUTICS, ET CETERA, ENCOURAGE YOU TO GO THERE. THIS IS A PRODUCT OF DAN AND HIS GROUP. THIS HAS CAUGHT THE INTEREST, WHOLE AREA CAUGHT THE INTEREST OF THE LAY PRESS AS WELL. POINTING OUT THE POTENTIAL FOR THESE COMPOUNDS TO DO NUMBER THERAPEUTIC THINGS INCLUDING CNS REMYELINATION AND MS. SO WE MADE IT IN 47 MINUTES OR SO. I'M GLAD TO TAKE ANY QUESTIONS THAT YOU HAVE. THERE WILL BE LOTS OF TIME LATER IN THE DAY FOR THAT AS WELL. BARRING THAT, WE HAVE A A BREAK UNTIL 10:30. DAN, I GUESS WE SHOULD GO UNTIL 10:30 BECAUSE THERE'S PROBABLY PEOPLE ON THE PHONE FOR THIS ONE. SO WE WILL TAKE A BREAK UNTIL 10:30 AND THEN RESTART WITH THE WORKING GROUP ON THE CTSA IOM REPORT PROMPTLY AT 10:30. WE HAVE A FULL TWO HOURS FOR THAT. THE PRESENTATION SHOULDN'T TAKE MORE THAN AN HOUR BUT WE REALLY WANT TO HAVE DISCUSSION AROUND THAT REPORT. AND WHERE WE GO FROM HERE. I LOOK FORWARD TO SEEING THE REPORT. BECAUSE THIS WILL BE THE FIRST TIME I WILL HAVE HEARD ABOUT IT TOO. SO LOOKING FORWARD TO THAT. BEFORE WE TAKE A BREAK ARE THERE ANY IMMEDIATE QUESTIONS? GOOD. ALL RIGHT. BE BACK AT 10:30. SO WE'RE NOW GOING ON TO A REPORT, I'M LOOKING FORWARD TO SEEING, AND HEARING ABOUT THAT IS THE RESULT OF A WORKING GROUP THAT WAS STARTED, LOOK FOR MY NOTES HERE. WHERE DID MY NOTES GO? SO THIS WORKING GROUP CAME FROM SOME DISCUSSIONS THAT WE HAD INTERNALLY AND I HAD WITH A NUMBER OF Y'ALL WHEN THE IOM REPORT ON THE CTSA PROGRAM CAME OUT LAST JUNE. YOU'LL REMEMBER IN THAT REPORT, MULTIPLE TIMES THE REPORT TALKED ABOUT THE NEED FOR A DESCRIPTION ON MEASURABLE OUTCOMES FOR THE PROGRAM BY WHICH INDIVIDUAL CTSAs INDIVIDUAL GRANTEES AND THE PROGRAM AS A WHOLE CAN BE MEASURED. SO WE GAVE -- WE PUT THIS WORKING GROUP TOGETHER TO HAMMIEST WITH THOSE MEASURABLE OUTCOMES BUT ALSO HELP US IN GENERAL WITH ACTUALLY MAKE REAL RECOMMENDATIONS OF THE REPORT, THE WAY ALAN LESHNER AND SHARON TERRY CO-CHAIRS YOU REMEMBER PART OF THE COMMITTEE PUT IT WAS THAT THEY FELT THE CHARGE WAS SO BROAD AND POTENTIALLY TRANSFORMATIONAL THEIR RECOMMENDATIONS WERE NECESSARILY AT THE 20 TO 30,000-FOOT LEVEL, WHAT NCATS NEEDS TO DO PROGRAM LEVEL SON GROUND, AT THE GROUND LEVEL IN THE PER VERBIAL WEEDS. SO WHAT WAS NEEDEDDED IS AN INTERMEDIATE STEP FROM 30,000 TO MAYBE 5,000, NOT THROUGH THE WEEDS BECAUSE THE WEEDS IS WHAT WE IMPLEMENT. BUT HELP US DEFINE IN MORE REDUCTION PRACTICE TERMS WHAT SOME THESE RECOMMENDATIONS MEANT. SO I GAVE THEM CHARGE TO DEVELOP MEANINGFUL MEASURABLE GOALS AND OUTCOMES FOR THE PROGRAM TO ADDRESS RECOMMENDATIONS OF THE REPORT, SPEAK TO CRITICAL ISSUES AND OPPORTUNITIES AS THEY SAW ACROSS THE FULL SPECTRUM OF CLINICAL TRANSLATIONAL RESEARCH. SO I'M GOING TO TURN THE FLOOR OVER NOW TO THE 3 HEADS OF CO-CHAIRS OF THE WORKING GROUP AND THREE CHAIRS WE CHOSE VERY CAREFULLY TO PRESAGE WE THOUGHT WERE THREE OF THE CRITICAL STAKEHOLDERS FOR NCATS IN GENERAL IS RON BARTEK, PATIENT ADVOCATE, RUNS THE ATAXIA RESEARCH ALLIANCE, NORA DISIS, A CLINICAL RESEARCHER, A CLINICAL TUMOR KNEWNOLOGIST UNIVERSITY OF WASHINGTON AS WELL AS THE PI OF THE CTSA THERE. AND SCOTT WEIR WHO IS ASSOCIATED WITH THE CTSA KANSAS, SPENT MOST OF HIS LIFE IN PHARMA AT MARY ANN LABS AND HEAD OF INSTITUTE FOR ADVANCING MEDICINE UNIVERSITY OF KANSAS. SO REALLY LOOKING FORWARD TO WHAT ALL OF YOU HAVE BEEN DOING. I HAVE BEEN -- THEY HAVE BEEN MEETING AT OUR PLACE SO I HAVE BEEN SEEING THEM GO BACK AND FORTH IN A HUGE AMOUNT OF -- BEEN LIKE BEING NEXT TO A BEAVER DAM BEING BUILT. A LOT OF ACTIVITY SO LOOK FORWARD TO HEARING WHAT THE BEAVER DAM LOOKS LIKE. SOON AS UNVEILED. >> MY NAME IS NORA DISIS, I'LL GIVE YOU AN OVERVIEW OF THE PROCESS WHAT HAPPEN THE GOALS WERE. FIRST I WOULD LIKE TO INTRODUCE BRIEFLY TO THE PEOPLE INVOLVED IN THIS PROCESS. YOU HEARD ABOUT RON AND MYSELF AND SCOTT. WE WERE ALSO JOINED BY AN BONNHAM, MATT DAVIS, DAVID DEMETS GARY GIB BONNES, BOB HAIRINGTON, PHIL LEE, LYNN MARKS, SHARON MILFRAM, LEWIS NUGLIA FER THAN DA RAYS AND BOB TEMPER, SO A DIVERSE GROUP OF PEOPLE WHO HAD A LOT TO SAY ABOUT THEIR INDIVIDUAL COMPONENTS AND OUR GOAL WAS TO BRING IT ALL TOGETHER AS A WHOLE. BEFORE I LAUNCH INTO THE INTRODUCTION I WOULD LIKE TO ACKNOWLEDGE A FEW OTHER PEOPLE. THIS REPORT REALLY DRAWS ON THE EXPERIENCE OF A LOT OF PEOPLE WHO WORKED WEREN'T IN THE ROOM, MANY PEOPLE WHO WERE ON THE COMMITTEE WENT AROUND AND REALLY FOUND A LOT OF IDEAS IN THEIR COMMUNITY, PARTICULARLY NCATS LEADERSHIP AND STAFF. WE HAD A LOT OF DISCUSSIONS WITH THEM WHAT THEIR EXPERIENCES WERE. WE EXPRESS APPRECIATION TO THE MEMBERS OF THE WORKING GROUP AS I SAID BEFORE, WE ALSO EXPRESS TO MEMBERS OF THE IOM WORKING GROUP. THAT IOM REPORT WAS THE BASIS BY FOR OUR DELIBERATIONS. I WANT TO ACKNOWLEDGE PHIL LEE'S SPECIAL ROLE IN THIS PROCESS. PHIL IS LEADER IN RESULTS BASED ACCOUNTABILITY, I WILL TALK ABOUT THAT IN A LITTLE BIT. HE GUIDED OUR DELIBERATIONS AND GAVE US A GREAT TOOL FOR THE ORGANIZATION OF THE REPORT. AND I WOULD ALSO LIKE TO ACKNOWLEDGE THAT THERE WERE PEOPLE WHO HELPED US PUT TOGETHER THE REPORT CHRISTINE CATULO AND SHERRY ANY NOLS, THEIR ABLE TO -- NICHOLS AND THEIR ABILITY TO BE THERE INVALUABLE. WE WOULDN'T BE ABLE TO PUT TOGETHER THIS REPORT UNLESS THERE WAS SOMETHING TO TALK ABOUT. WE REALLY WANTED TO THANK ALL THE MEMBERS OF THE CTSA PROGRAM ACROSS THE NATION. BECAUSE THEY'VE DONE A REMARKABLE JOB AS WE SAT AND THOUGHT ABOUT THE POSITIVE AND THE CHALLENGES OF THE PROGRAM, THERE WAS AN AWFUL LOT TO DISCUSS. SO WE HOPE THIS REPORT WILL BE VERY USEFUL FOR THEM AS A GUIDANCE. SO WHAT WAS OUR CHARGE? OUR CHARGE WAS TO DEVELOP MEANINGFUL MEASURABLE GOALS AND OUTCOMES FOR THE CTSA PROGRAM. THAT ADDRESS RECOMMENDATIONS OF THE IOM REPORT. AS CHRIS SAID NOT TO MAKE METRICS OF THE PROGRAM, THAT WILL COME LATER. WE'RE TALKING MEASURABLE OUTCOMES. ANOTHER GOAL WAS TO SPEAK TO THE CRITICAL ISSUES AND OPPORTUNITIES ACROSS THE SPECTRUM OF CLINICAL TRANSLATIONAL RESEARCH. WHAT ARE THE UNIQUE THINGS WE COULD DO TO MAKE IMPACT? LET ME TELL YOU OUR TIME LINE, IT WAS AGGRESSIVE. WE HAD OUR INITIAL FACE TO FACE MEETING EARLY GUESS DECEMBER 2013. WEED HAD A TELECONFERENCE IN JANUARY. OUR SECOND FACE TO FACE MEETING WAS IN FEBRUARY. WE HAD A DRAFT REPORT OUTLINED BY MARCH AND DRAFT REPORT BY APRIL AND HERE WE ARE MAY 16th. SO IT WAS A VERY INTENSIVE PROCESS WITH A LOT OF COMMUNICATION AND HOPE YOU WILL SEE REFLECTED IN A REASONABLE PRODUCT THAT'S UP TO YOU. HOW DID WE IMPLEMENT THE IOM REPORT RECOMMENDATION? LET ME GIVE JUNIOR VIEW NOT ONLY OF THE PROCESS WE WENT THROUGH BUT WHAT WE THINK THE PROCESS GOING FORWARD WILL BE. AS EVERYONE KNOWS THE IOM REPORT CAME OUT IN JUNE OF 2013. OUR GROUP SO SET THE STRATEGIC GOALS AND IDENTIFY MEASURABLE OBJECTIVE, THIS IS MAY OF 2014. NCATS THROUGH THE U 54 MECHANISM WILL DEVELOP AN IMPLEMENTATION STRATEGY AND PROGRAM MA METRICS. THAT MAYBE APPLIED ACROSS THE CTSA PROGRAM. AS THESE ARE IMPLEMENTED AND NCATS CAN THEN USE THE METRICS TO MEASURE RESULTS. SO THAT IS THE BIG VISION OF THE BEINGS OF THIS REPORT. IOM RECOMMENDATIONS ARE KITS STILLED DOWN TO SEVEN MAIN POINTS. THE FIRST THREE WERE TO STRENGTHEN NCATS LEADERSHIP OF THE CTSA PROGRAM. TO RECONFIGURE AND STREAMLINE THE CTSA CONSORTIUM. AND THEN TO BUILD ON THE STRENGTH OF THE INDIVIDUAL CTSA ACROSS THE SPECTRUM OF RESEARCH. THOSE THINGS WERE NOT THINGS THE WORKING GROUP COULD TACKLE. THESE ARE REALLY ISSUES INTERNAL AND COULD BE ADDRESSED BY THE NCATS STAFF. SO THAT WAS MOVED OFF THE WORKING GROUP PLATE. THAT LEFT US WITH FOUR REMAINING RECOMMENDATIONS FORMALIZE STANDARDIZE EVALUATION PROCESS, AS YOU CAN SEE WE HOPE THIS IS THE BEGINNINGS OF THAT, TO ADVANCE INNOVATION AND EDUCATION AND TRAINING PROGRAMS, ENSURE COMMUNITY ENGAGEMENT IN ALL PHASES OF RESEARCH. AND STRENGTHEN CLINICAL TRANSLATIONAL SCIENCE RELATIVE TO CHILD HEALTH. THESE WERE ELEMENTS CONSIDERED BY THIS ADVISORY GROUP IN TERMS OF PUTTING TOGETHER THIS REPORT. SO I HAVE THOSE FOUR IOM RECOMMENDATIONS I JUST TALKEDDED ABOUT. WHAT WE DID WITH OUR FIRST MEETING IS WE REALLY LOOKED AT SPECIFIC FOCUS AREAS, BECAUSE MANY RECOMMENDATIONS FROM THE IOM REPORT HAD INTERLOCKING AND OVERLAPPING TOPICS. SO WE FIRST STARTED BY TRYING TO GET TOPICS INTO DISCRETE CATEGORIES. FOCUS AREAS WE DISCUSSED WERE TRAINING AND EDUCATION, COLLABORATION AND PARTNERSHIP, ESPECIALLY DIVERSITY, COMMUNITY ENGAGEMENT OF STAKEHOLDERS. IF ACADEMIC ENVIRONMENT FOR TRANSLATIONAL SCIENCE WAS AN ISSUE AND TRANSLATIONAL SCIENCE ACROSS THE LIFE SPAN, IT WAS NOT ONLY CHILDREN BUT SPECIAL POPULATIONS AND OTHER PEOPLE OR GROUPS LEV OUT OF THE TRANSLATIONAL SCIENCE RUBRIC. AS WE TALK ABOUT THOSE WE KEPT SEEING LOTS OF ENTERLAP AND OVERLAP SO WE FIRST DISTILLED THOSE TO STRATEGIC GOALS DISCREET FROM ONE ANOTHER THOSE STRATEGIC GOALS WERE WORK FORCE DEVELOPMENT, COLLABORATION AND ENGAGEMENT, INTEGRATION, AND METHODS AND PROCESSES SO NOW WE HAD FOUR AREAS THAT WE COULD HONE IN DISCUSS PROS AND CONS AND IDENTIFY MEASURABLE GOALS. THESE STRATEGIC GOALS START OUT WITH A STRATEGICCAL STATEMENT SO WORK FORCE DEVELOPMENT, WE WERE LOOKING FOR TRANSLATIONAL SCIENCE WORK FOES THAT WE ARE LOOKING TO ADVANCE TRANSLATION. THIS WAS REALLY IN RESPONSE TO ANY STAKEHOLDER AT THE TABLE SHOULD BE ENGAGED. NOT JUST AT THE TABLE. FOR INTEGRATION WE LOOK AT TRANSLATIONAL SCIENCE INTEGRATED ACROSS ALL PHASES ACROSS THE LIFE SPAN SO THAT IN PEDIATRICS IT'S NOT ALL ABOUT DISSEMINATING ADULT DRUGS. AT EVERY PHASE THERE HAS TO BE DISCOVERY N GERIATRIC MEDICINE THERE'S DISCOVERY, THAT EVERY POPULATION WOULD BENEFIT FROM ALL PHASES OF TRANSLATIONAL RESEARCH. FINAL METHODS AND PROCESSES THAT THE CTSA, THIS IS THE SWEET SPOT THAT IT EMBRACES THAT THE SCIENTIFIC STUDY OF PROCESS CONDUCTING TRANSLATIONAL SCIENCE IN AND OF ITSELF ENABLED SIGNIFICANT ADVANCES IN THE TRANSLATION WE COMMENTED EACH INDIVIDUAL CTS ACTSES AS A LIVING LABORATORY THE SOLVE THE PROBLEMS THAT ARE HOLDING TRANSLATIONAL MEDICINE BACK. NOW, THESE ARE GREAT IDEAS BUT HOW DO WE ORGANIZE THESE? FOR THIS, WE REALLY USE THIS TOOL CALLED RESULTS BASED ACCOUNTABILITY AS A DIAGNOSTIC TOOL AND AS A DISCUSSION. SO WHAT THE TOOL REQUIRES IS THAT YOU START OUT WITH A STRATEGIC GOAL STATEMENT, THE BROADER GOAL FOR TRANSLAT? AL SCIENCE WITHIN THE CTSA PROGRAM, AND THEN WE DID A DIAGNOSTIC, WE LISTED AND PRIORITIZED THE FACTORS THAT IS I DRESSED WOULD RESULT IN GREATEST PROGRESS TOWARD ACHIEVING THE STRATEGIC GOALS. SO THERE ARE POSITIVE FACTORS AND THEN NEGATIVE FACTORS OR CHALLENGES OR HOLES OR GAPS WE NEEDED TO TACKLING. FINALLY WE END UP WITH A MEASURABLE OBJECTIVE. THESE MEASURABLE OBJECTIVES WOULD ACT AS A REFERENCE OR A GUIDE FOR THE CTSA PROGRAM. THAT PEOPLE COULD USE IT IN DESIGNING, IMPLEMENTING ENGAGING THE ROLE SUCCESS OF THE CTSA WILL PLAY CONTRIBUTING TO THE ACHIEVEMENT OF EACH INDIVIDUAL STRATEGIC GOAL. THIS IS NOT COMPREHENSIVE. BUT IT'S MEANT TO GIVE THE FLAVOR OF BIG HAIRY PROBLEMS WE THINK CAN BE TACKLED BY THE CTSA PROGRAM AND IN FACT ALL OF NCATS.. WHAT ELEMENTS WE TALKED ABOUT FOR EACH STRATEGIC GOAL AND WHAT IS PRESENTED HERE? WE FIRST CONSIDER THE ISSUES BUT IN THE PRESENTATION SCOTT WILL ONLY GIVE .4 THEN WHAT DOES SUCCESS LOOK LIKE? AND THEN WE TALKEDDED ABOUT WHAT FACTORS WILL IMPACT SUCCESS AND THEN THE MEASURABLE OBJECTIVES ARE WHAT WILL IT TAKE TO GET THERE? SO WE REALLY HOPE THIS REPORT ACTS AS A ROAD MAB OF STEPS TO GET THERE AND FROM THOSE STEPS CHRIS AND NCATS STAFF CAN DRILL DOWN TO IMPLEMENTATION AND HOW YOU MEASURE THAT. WITHOUT ANY FURTHER ADIEU I WOULD LIKE TO INTRODUCE RON BARTEK, WHO WILL GO OVER THE FIRST TWO STRATEGIC GOALS OUT OF THE FORM. THANKS, RON. >> THANK YOU, NORA. I WOULD LIKE TO SAY FIRST WHAT A PRIVILEGE IT'S BEEN TO WORK WITH NORA AND SCOTT AND THE OTHER TALENTED PEOPLE ON THE WORKING GROUP, IT'S AN AN EXPERIENCE FOR ME, THANKS THE NCATS FOR THE OPPORTUNITY ALSO FURTHER TO SET THE STAGE TO AVOID, BECAUSE AS NORA SAID SCOTT AND I WILL GO THROUGH THE NITTY GRITTY OF THE REPORT IN TERMS OF WHAT IT WILL TAKE TO GET TO THE END POINTS WE HAVE IDENTIFIED. TO AVOID MAKING THAT SUCH A PLATITUDENESS AND BORING RECITATION OF FACTS, I WOULD LIKE TO SET THE STAGE A LITTLE BIT IN THE POLITICAL CONTEXT THAT CHRIS AND MARGARET POSED THIS MORNING, THAT IS BECAUSE I THINK THIS POLITICAL ENVIRONMENT OFFERS A REAL OPPORTUNITY TO BREATHE LIFE INTO THIS REPORT AND TO CHANGE THESE STRATEGIC GOALS AND MEASURABLE OBJECTIVES AND THEYS WE WILL GET THERE INTO REAL AND REALISTIC OBJECTIVES. I SAID THAT ON THE STRENGTH OF THINGS CHRIS COLD YOU ABOUT IN TERMS OF CONGRESSMEN UPTON AND DEGETS RECENTLY ANNOUNCED INITIATIVE 21st CENTURY CURES ACT. WE DON'T KNOW HOW THIS MOVIE IS GOING TO END. AS MARGARET INDICATED THIS MORNING. CHRIS AS WELL. BUT WE KNOW LIT BE A 3-D MOVIE. NCATS IS FOND OF 3-D MOVIES. CONGRESSMAN UPTON HAS A DIFFERENT SET OF 3DS BUT CLOSELY RELATED. HIS ARE DICOVERY, DEVELOPMENT AND DELIVERY SIMILAR TO NCATS 3. SHE IS PASSIONATE. HE IS THE REAL DEAL. I HAD THE FORTUNATE OPPORTUNITY TO HAVE DINNER WITH HIM LAST WEEK AND NORA GAYLA. I CAN REPORT TO YOU WITHOUT RESERVATION AS I KNOW MARGARET AND CHRIS CAN DO AND PROBABLY MANY OTHERS HERE IN THE ROOM THIS IS A GENTLEMAN GENERAL ONELY COMMITTED TO OUR CAUSE, GENUINELY COMMITTED TO IMPROVING THE HEALTH OF THE UNITED STATES OF AMERICA. THIS INITIATIVE WAS,5/ FIRST STAGE WAS SET FOR IT, THE ROUND TABLE CHRIS MENTION AD FEW DAYS AGO. IN WHICH MARGARET PARTICIPATED, FRANCIS COLLINS LED THE PANEL DISCUSSION. FRANCIS COLLINS WAS VERY ADAMANT, HE SHOWED THE CHIP, THE TISSUE CHIP AND WON'T SAY TESTIMONY NIH IS NOT ASKING FOR HAND OUT, IT'S NOT ASKING FOR A DONATION TO MEDICAL SCIENCE, IT'S ASKING FOR INCREASED INVESTMENT IN THE FUTURE OF THE UNITED STATES OF AMERICA'S HEALTH AND ECONOMY. THAT HELP SET THE STAGE FOR OTHER TESTIMONY FROM HIS COLLEAGUES. MARGARET AMONGST THEM AND SPOKE COMPELLINGLY, LET HER TAKE A MINUTE TO GIVE HER QUICK ASSESSMENT OF HER INTERVENTION. >> I PICKED UP ON CHRIS'S NUMBERS. THAT SEEMED TO HAVE RESONATE FORD FOLKS THAT WE HAVE 7,000 DISEASES AND AROUND 500 TREATMENTS. TO SET THE STAGE, OFTEN PARTICULARLY GIVEN CHRONIC DISEASE EPIDEMIC THAT FACE IT IS UNITED STATES AND CERTAINLY THE WORLD, THERE CAN BE PUSH BACK WHY WE'RE WORRIED ABOUT NEW CURES AND TREATMENTS WHEN THERE'S SO MUCH PREVENTION WORK THAT NEEDS TO BE DONE AND OBVIOUSLY WE WANT TO SEE AN ECOSYSTEM THAT ALLOWS BOTH. WE NEED TO PREVENT IF WE CAN PREVENT. BUT FROM THERE IS THE UNFORTUNATE CHANCE THAT YOU CAN BE DIAGNOSED WITH SOMETHING TOMORROW, THERE'S JUST NOT MUCH IN THE PIPELINE FOR YOU. RIGHT NOW THEY'RE THE TOP OF THE FUNNEL AND THEY'LL WHITTLE DOWN, BOB BELL AND I HAVE DISCUSSIONS AT THE BREAK AROUND POINTS WHICH THIS EFFORT CAN CRYSTALLIZE AND GET NARROWED BECAUSE I THINK THERE ARE A NUMBER OF PLACES THEY CAN GO. I AGREE IN TERMS OF REPORT, THERE IS OPPORTUNITY. THAT WAS ONE OF THE POINTS THAT I RAISED AND OTHER REPRESENTATIVES MAKING STRONGLY, THERE IS THE POSSIBILITY IN TERMS OF WORK FORCE IF WE TAKE OUR FOOT OFF THE PETAL TOO LONG THERE'S NOT A WORK FORCE THERE WHEN WE GO BACK TO IT IF YOU DON'T KEEP IT STRONG AND VIBRANT. FROM IS WORK TO BE DONE IN UNDERSTANDING THE CONTINUUM, FEEDING THE BASIC SCIENCE AND HAVING THINGS TO ACTUALLY BE ABLE TO TRANSLATE. I THINK THERE'S ANY NUMBER OF PLACES THEY CAN GO BUT HAVING LITERALLY GOTTEN OFTEN A PLANE FROM EUROPE YESTERDAY AND EVERYTHING IN THIS ROOM KNOW IT IS REST OF THE WORLD ISN'T SITTING AROUND TWIDDLING THEIR FINGERS WONDERING IF THEY SHOULD OR SHOULDN'T CARE ABOUT LIFE SCIENCES, THEY CARE DEEPLY, THEY ARE PRAGMATIC ABOUT IT AND THIS IS A UNIQUE OPPORTUNITY IN THE U.S. TO PUT FOCUS BACK ON WHAT ARE CHALLENGES AND DO P SOME LEADING AROUND SOLUTIONS. BOB I DON'T KNOW IF DO YOU WANT ADD ANYTHING TERMS OF PRECISION MEDICINE YOU TALK ABOUT. >> WHERE WE WOULD LIKE TO THE SEE THE FDA MOVE TO BECAUSE WE ALL MOVE TO ISSUE OF PERSONALIZED MEDICINE. IF THAT'S THE MEDICINE OF THE FUTURE BUT MANY OF US NOT SURE AT THIS POINT THAT THE FDA IS READY FOR PERSONALIZED MEDICINE. THEY TALK ABOUT THE TOP BUT NOT AT THE LEVEL OF THE DIVISIONS FOCUSED ON THAT. THE CHALLENGE IS TO GET DISCUSSIONS FOCUSED. LET' TALK ABOUT THAT, THAT IMPACTS ON US IN THIS ROOM AND IF THERE'S A WAY THIS CAN HAPPEN. UNLESS THE DIVISIONS ARE STARTING TO THINK ABOUT THE, YOU CANNOT DO A PLACEBO CONTROLLED TRIAL ON MUTATION THAT ONLY EFFECTS SIX PEOPLE, IT WON'T HAPPEN. WHAT HAPPENS WITH INDUSTRY AND WHAT'S GOING TO HAPPEN WHEN WE HAVE THESE GREAT OPPORTUNITIES IN GENETIC MEDICINE. THAT'S WHAT WE WERE FOCUSING TRYING TO GET THE COMMITTEE AND EFFORT TO FOCUS. THAT YOU WILL SEE WOVEN THROUGHOUT OUR PRESENTATION TODAY, I RECOGNIZE TOO ONE OTHER POINT MARGARET RAISED COMPELLINGLY AT THE ROUND TABLE WAS THE IMPORTANCE OF ANOTHER STAKE HOLE E THAT IS THE PATIENT. GETTING THE PATIENT TO THE TABLE GENUINELY, NOT JUST PRO FORMA I SERVE ON THIS COMMITTEE LET'S GO FOR IT BUT GENUINELY GETTING THE PATIENT AND THE PATIENT ADVOCATE GENUINELY AT THE TABLE FROM THE GET GO. THROUGHOUT THE ENTIRE PROCESS IS REALLY IMPORTANT. TO THIS POINT MARGARET AND BOB AT THE FDA, RECOGNIZING THE FDA COLLEAGUE WITH US, LIKE TO RECOGNIZE THE IMPORTANT INTERVENTION IN MY VIEW OF JANET WOODCOCK AT THE SAME ROUND TABLE DISCUSSION, WHEN SHE WAS CHALLENGED ABOUT THE SPEED OF WHY DOES IT TAKE TEN TO 15 YEARS TO GET A DRUG TO THE CLINIC. SHE SAID LET'S FIRST NOT ALWAYS FOCUS ON TRYING TO ACCELERATE THE APPROVAL PROCESS. WE PROBABLY ARE GETTING CLOSE TO THE NUMBER ON THE APPROVAL -- NUB ON THE APPROVAL PROCESS, MIGHT BE GET MUCH BETTER FOR THREE MONTHS TO APPROVE CALIDACO FOR CYSTIC FIBROSIS THERE'S FOCUS TON DEVELOPMENT, SEE HOW WE CAN ACCELERATE THAT, RATHER TAKING 10 TO 15 YEARS TO GET THE DRUG, THERAPY, THAT INTERVENTION TO THE FDA. LET'S SEE IF WE CAN GET INSIDE THAT AND WHAT JANUARY SAID TO THAT POINT, REMARKABLY APROPOS TO THIS PRESENTATION TODAY WAS WE NEED TO HAVE BETWEEN THE FDA REVIEWERS AND THE DRUG DISCOVERER, WE NEED TO HAVE AN EFFECTIVE CLINICAL NETWORK THAT HELPS THAT DISCOVERY KNOW HOW TO DO AN EFFECTIVE MORE ACCELERATED CLINICAL TRIAL AND GET THE PRODUCT DEMONSTRATED WHEN -- AND GET TO IT THE FDA AND IN A LOT QUICKER FASHION. I SORT OF STOOD UP AND CHEERED INTERNALLY TO HEAR THAT. BECAUSE WHAT BETTER COLLABORATIVE EFFECTIVE CLINICAL NETWORK DO WE KNOW ABOUT OTHER THAN THE CTSA? TO DO CHAIRMAN UPTON 3-D DISCOVERY, DEVELOPMENT, AND DELIVERY. IT'S -- WE HAVE THE NETWORK. WE NEED TO MAKE IT WORK. BETTER. AND THERE'S NOBODY HERE THAT WOULD ARGUE WITH THE IOM REPORTS SUGGESTION THINGS OF THE C THETSA PROGRAM, WE NEED A MORE EFFECTIVE COLLABORATIVE CLINICAL NETWORK. WITH THAT LENGTHY MIND SET LET'S GET RIGHT TO THE STRATEGIC GOALS. YOU HEARD NORA SAY WE CAME ONE FOUR FOCUS AREAS. THEY'RE NOT INTENDED TO BE ONE TO ONE TRANSLATIONS FROM THE FOUR RECOMMENDATIONS OF THE IOM REPORT. WITH WHICH WE'RE CHARGED. RATHER THEY ARE AS NORA DESCRIBED OUR EFFORT TO COME UP WITH FOUR GOALS THAT WOULD HELP INSTRUCT ALL THE NCATS EFFORTS TO MEET ALL SEVEN RECOMMENDATIONS OF THE IOM REPORT AND MEASURABLE OBJECTIVES AND TACTICAL OBJECTIVES THAT NEED TO BE ACCOMPLISHED TO GET THERE. THE FIRST FOCUS AREA WAS WORK FORCE DEVELOPMENT. AND THE STRATEGIC GOAL WE SET FOR THAT WAS TO HAVE TRANSLATIONAL SCIENCE WORK FORCE WITH THE SKILLS AND KNOWLEDGE NECESSARY TO ADVANCE TRANSLATION HAND DISCOVERIES. YOU SEE HERE ARE INITIAL CONSIDERATIONS INCLUDED THAT OF THE KNOWLEDGE THAT HEALTH NEEDS OF OUR NATION CALL FOR SCIENTIST TRAINED TRANSLATIONAL INFORMATIVE SCIENCE WITH LEADERSHIP AND TEAM SKILLS TO ENGAGE IN EFFECTIVE COLLABORATIONS. WE KNEW THE WORKING GROUPS EARLY DISCUSSIONS WERE FORMED AROUND IS THAT GLASS HALF FULL OF HALF EM THETY? WE CONCLUDED THAT IT IS AT LEAST HALF FULL. YOU WILL SEE EVIDENCE OF THAT CONCLUSION IN THE PRESENTATIONS THAT'S GOD GOT AN IDEA. THE TRANSLATIONAL FIELD IS DEFINITELY GROWING WORLDWIDE BASIS. SEASONED AND NEWLY EDUCATED PROFESSIONALS ARE SEEKING OPPORTUNITIES IN THAT SPACE, GROWING RECOGNITION OF TRANSLATIONAL SCIENCE AS HIGHLY VALUED CAREER PATH IS EXTREMELY IMPORTANT IN THIS EFFORT. AND DEMAND FOR TRAINED PROFESSIONAL WHOSE SPECIALIZE IN TRANSLATIONAL SCIENCE IS GROWING. HOW FAR THE GLASS IS LESS THAN HALF FULL BECAUSE THERE'S STILL THE NEED FOR BETTER DEFINITION OF THE DISCIPLINE, INNOVATIVE APPROACHES TO TRAINING. NEW BENCH MARKETS FOR TEAM BASED RESEARCH AND EDUCATIONAL OPPORTUNITIES FOR EVERYONE IN TRANSLATIONAL RESEARCH TEAM. SO WHAT WILL IT TAKE TO GET THERE, WHAT WILL SUCCESS LOOK LIKE? HOW WILL WE KNOW WE'RE THERE TO ACCOMPLISH THAT STRATEGIC OBJECTIVE FOR THE WORK FORCE? THE WORK GROUP BELIEVES THAT SPACE WILL LOOK LIKE AN ECOSYSTEM IN WHICH TRANSLATIONAL SCIENCE IS USED VIEWED AS THE PLACE TO GO. NO LONGER DISTANT COUSIN BUT THE PLACE TO GO. BY THOSE WHO WANT TO PURSUE HIGH IMPACT CAREERS IN HEALTH SCIENCES. LOOKED LIKE AN ECOSYSTEM THE TRANSLATIONAL SCIENCE WORK FORCE MEETS THE RECOGNIZED NEEDS OF TODAY. AND EMERGING NEEDS OF TOMORROW AND THAT DEFINES RESEARCHERS PRACTITIONER, PATIENT, PATIENT ADVOCACY ORGANIZE, INDUSTRY, COMMUNITY MEMBERS AND GOVERNMENT AGENCIES. THE CURRICULA REQUIRED WILL NEED TO TRAIN A WORLD--LEADING GLOBALLY CONNECTED CONTINUALLY LEARNING WORK FORCE WITH THE SKILLS TO EXCEL AND TRANSLATIONAL SCIENCE. AND THAT -- THOSE SORTS OF CURRICULA HAVE BEEN ESTABLISHED. THE SUBDISCIPLINES WITHIN TRANSLATIONAL SCIENCE WILL HAVE TO BE FAR BETTER DEFINED AND STEPS TO ACHIEVEMENT IN ADVANCEMENT WITHIN EACH TRANSLATIONAL SCIENCE DISCIPLINE WILL HAVE TO BE FAR BETTER DEFINED. WHAT POSITIVE AND NEGATIVE FACTORS THAT WENT INTO WHETHER THE GLASS WAS HALF FULL OR HALF EMPTY? HERE THEY ARE LISTED. I'LL HIGHLIGHT A FEW. THE POSITIVE FACTORS INCLUDED THAT A SIGNIFICANT NUMBER OF INTERESTED PEOPLE WANT TO ENGAGE IN TRANSLATIONAL SCIENCE. THE WORLD WAY DEMAND IS INCREASING. THERE ARE MORE AND MORE DIVERSE CAREER PATHWAYS. THERE'S A GREAT DEAL OF PROGRESS IN THE CTSAs TO DATE AND THE NEW TECHNOLOGIES ARE ENABLING US TO ESTABLISH GLOBAL NETWORKS OF EDUCATION. THE NEGATIVE FACTORS ON THE OTHER HAND INCLUDE FACT THERE ARE NO CLEAR COLLECTIVE STRATEGIES FOR FUNDING OR DEVELOPING TRANSLATIONAL WORK FORCE. TRANSLATIONAL WORK FORCE HAS IN MOVING PARTS. THE CURRICULA ARE FAR TOO GENERAL AND MAKES IT VERY HARD TO RECOGNIZE TRANSLATION HAL SCIENCE BASE AS LEGITIMATE VALUE SPACE. THE SUBDISCIPLINE SKILLS ARE NOT DEFINED THE WORK FORCE STRATEGIES SHOULD NOT INVOLVE ALL THE KEY STAKEHOLDERS AND TRANSLATIONAL SCIENCE TEAM MEMBERS ARE NOT EQUALLY VALUED. SO WHAT MEASURABLE OBJECTIVES MIGHT WE CONSIDER TO GETTING TO THAT FROM WHERE WE ARE NOW TO THAT ACHIEVING THAT STRATEGIC OBJECTIVE OF THAT ECOSYSTEM THAT HAS ALL THOSE CREDIBLE OUTCOME? WE FIRST DECIDED THE CORE COMPETENCIES FOR TRANSLATION HAL SCIENCE -- AND THE METHODS FOR EVALUATING THOSE COMPETENCIES HAVE TO BE WELL DEFINED. I'M GOING TO SKIP THROUGH THE REST OF THAT AND GIVE YOU THE HIGHLIGHTS AS NORA ENCOURAGED US TO DO. AND THE SECOND MEASURABLE OBJECTIVE IS TRANSLATIONAL SCIENCE BECOME AS FULLY DEVELOPED HIGHLY VALUE AND REWARDING DISCIPLINE WITH FORMAL CAREER PATHWAYS. ESPECIALLY IN TERMS OF TRANSLATIONAL SCIENCE ORGANIZATIONAL UNITS HAVING BEEN ESTABLISHED IN ACADEMIC HEALTH CENTERS AND THEY HAVE THE SAME STATURE A OTHER BIOMEDICAL DEPARTMENTS IN THE SAME INSTITUTIONS. THIRD, THAT TRANSLATIONAL SCIENCE BECOMES A A FULLY DEVELOPED HIGHLY VALUED AND REWARDING DISCIPLINE WITH FORMAL CAREER PATHWAYS. LIKE THE TO HIGHLIGHT THERE THE SYSTEMS FOR STAFF DEVELOPMENT AND FACULTY PROMOTION AT ACADEMIC HEALTH CENTERS WOULD THEN RECOGNIZE AND REWARD COLLABORATIVE NATURE OF TRANSLATIONAL SCIENCE. FOURTH TRANSLATIONAL SCIENCE IS CONDUCTED BY INTERDISCIPLINARY TEAMS AND I THINK WE HAVE TO EXPAND ON THIS. THERE ARE MEANS TO DEFINE RECOGNIZE AND REWARD THE CONTRIBUTIONS OF ALL SUCH TEAM MEMBERS, THERE ARE SUSTAINABLE PARTNERSHIPS IN THIS SPACE, BECOME THE NORM RATHER THAN EXCEPTION THE REWARD SYSTEMS THESE INSTITUTIONS ARE BUILT AROUND TEAM SCIENCE METRICS. AND THAT INDIVIDUAL TEAM MEMBERS ARE VALUED WITH CLEAR CAREER PATHS. TRANSLATIONAL WORK FORCE IS TRAINED IN CO-COMPETENCIES OF COLLABORATION AND ENGAGEMENT. FIFTH, CO-COMPETENCIES ARE AVAILABLE TO ALL MEMBERS OF THE WORK FORCE THROUGH VEHICLES LEARNING THAT ARE READILY ACCESSIBLE AND FLEXIBLE. SIX, TRAINING CURRICULA ON EFFECTIVE TEAMS HIGH PERFORMING ORGANIZATIONS AND PRODUCTIVE COLLABORATIONS ARE DEVELOPED. MAY BROADLY AVAILABLE AND VIEW AS CORE ELEMENT OF SUCCESS. I WILL GO TO THE NEXT FOCUS AREA FOR OUR GROUP ONE OF MY FAVORITES, THE IMPORTANT -- IMPORTANCE OF COLLABORATION AND ENGAGEMENT. THE STRATEGIC OBJECTIVE WE SET THERE WAS THAT STRATEGIC GOAL WAS STAKEHOLDERS ARE ACTUALLY ENGAGED, GENUINELY ENGAGED IN COLLABORATIONS TO ADVANCE TRANSLATION. OUR KEY CONSIDERATIONS IN THIS CATEGORY WERE THAT THE BENEFITS OF CLAN RATION ENGAGEMENT OBVIOUSLY INCLUDE STRONGER COMMUNITY SUPPORT AND FUNDING, ATTRACTING YOUNG PEOPLE AND CAREERS, TO CAREERS IN TRANSLATIONAL SCIENCE, FAR GREATER INNOVATION AND MORE ROBUST TRANSLATIONAL SCIENCE ENTERPRISE INCLUDING MORE TIME AND SUCCESSFUL CLINICAL TRIALS. WE ALSO RECOGNIZE WITHIN THE CTSA PROGRAM COLLABORATIVE PARTNERSHIPS ARE OFTEN LIMITED TO ACADEMIC INDUSTRY COLLABORATORS WITH SELECT COLLABORATIONS FROM THE COMMUNITY KNOWING THAT'S NOT SUFFICIENT. FINALLY I'LL EMPHASIZE DIVERSE STAKE HOLDERS PROVIDE UNIQUE VALUABLE VIEW POINTS AND MUST BE THOUGHTFULLY INCLUDED AND GENUINELY INCLUDED TO ACCELERATE TANGIBLE HEALTH IMPACTS. THOSE STAKEHOLDER INCLUDE PATIENTS, COMMUNITY PROGRAMS, COMMUNITY HEALTH PRACTICES, GOVERNMENT AGENCY, COMMUNITY HEALTH PRACTICES AND NON-PROFIT ORGANIZATIONS. JUST A WORD ON THE SIDE ABOUT NON-PROFIT ORGANIZATIONS. HE IS ARE NOT FATHERS OR GRANDFATHERS NON-PROFIT ORGANIZATIONS, IT'S ANY NUMBER OF PEOPLE HERE AT THE TABLE CAN TELL YOU, THESE CONTEMPORARY PATIENT ORGANIZATIONS AND SOME ARE WELL REPRESENTED HERE ON COUNCIL, ARE NOT LIKE THEY WERE IN THE PAST. THESE ARE NOT DESPERATE PARENTS TELLING YOU YOU NEED TO DEVELOP A THERAPY FOR THEIR KIDS. THESE ARE VERY SOPHISTICATED ORGANIZATIONS NOW WHO CAN HELP EACH CTSA SITE AND HELP THE CTSA PROGRAM WITH SUCH FAR-RANGING THINGS AS HAVING FULLY DEVELOPED NATURAL HISTORY STUDIES, AND DISEASE GROUP, HAVING FULLY DEVELOPED PATIENT REGISTRIES OF DISEASE GROUPS, HAVING CLINICAL NETWORKS WELL TRAINED WITH CLINICIANS WHO SEE THEIR PATIENTS EVERY YEAR AND HAVE PLENTY OF LONGITUDINAL DATA. THESE ARE PATIENT ORGANIZATIONS THAT CAN HAVE A DRUG COMPANY COME AND SIT AT THEIR TABLE WITH KEY OPINION LEADERS. AND DESIGN PROTOCOLS, THEY CAN MINE NATURAL HISTORY DATABASES TO DESIGN THEIR CLINICAL TRIALS AND COME UP WITH FAR BETTER CLINICAL TRIAL DESIGNS, THEN THEY CAN GO OUT AND RECRUIT THOSE CLINICAL TRIALS. ONE ANECDOTAL PIECE OF EVIDENCE THERE, OUR FOUNDATION RECRUIT AD PIVOTAL STUDY IN OUR DISEASE, THREE SITES, 60 PATIENTS AND TWO HOURS 43 MINUTES. AS CONTRASTED WITH THE YEARS IT TAKES OFTEN, THIS IS A RARE DISEASE. YEARS IT TAKES MOST OF YOU KNOW BETTER THAN I THAT MOST CLINICAL TRIALS FAIL AND MOST THAT FAIL DO SO BECAUSE INSUFFICIENT PATIENT PARTICIPATION. THAT NO LONGER HAS TO HAPPEN. SO JUST ENCOURAGING YOU ALL TO CONSIDER HOW IMPORTANT IT WILL BE FOR THE INDIVIDUAL CTSA SITES AND THE CTSA PROGRAM AT LARGE TO MAKE FULL USE OF THESE VERY EAGER PATIENT ADVOCATES. (OFF MIC) THREE SITES, 60 PATIENTS, WE RECRUITED FULLY, IT WAS FULLY RECRUITED IN TWO HOURS 43 MINUTES. THAT SAYS A LOT ABOUT THE EAGERNESS OF OUR PATIENTS OBVIOUSLY. WHAT SOPHISTICATION OF PATIENT FAMILIES. EFFICACY ORGANIZATIONS THAT ARE READY, WILLING AND EAGER TO SERVE. [APPLAUSE] HOW DO I GO AFTER THAT? WHAT DOES SUCCESS LOOK LIKE IN THAT COLLABORATION ENGAGEMENT SPACE IN THE STRATEGIC GOALS WE SAID THERE WAS FOR AN AN ECO AS I SAID IN WHICH FOLLOWING WOULD TAKE PLACE. COLLABORATIVE TEAM SCIENCE BECOME IT IS THE NORM RATHER THAN EXCEPTION. DIVERSE STAKEHOLDERS BECOME GENUINELY ENGAGED. AND COMMUNITIES INVOLVED IN ALL ASPECTS OF TRANSLATIONAL SCIENCE FROM THE VERY GET GO TO FINAL ANALYSIS, THEY'RE THERE AT THE TABLE AND HELPING. NOT JUST PRO FORMA ENGAGED. THE VALUE OF COLLABORATION IS ENHANCED WHY WOULD COST AND DIFFICULTIES OF COLLABORATION ARE REDUCED. TRANSLATIONAL SCIENCE IS GOVERNED BY COLLABORATIONS AND PARTNERSHIPS THAT REWARD ALL STAKEHOLDERS. RESEARCHERS, PATIENTS, INDUSTRY, COMMUNITY. PATIENT ADVOCATE COMMUNITY MEMBERS AND CITIZEN SCIENTISTS HAVE TOOLS AND INFRASTRUCTURE IN PLACE TO PARTICIPATE FULLY AS PARTNERS IN ALL PHASES OF TRANSLATIONAL SCIENCE AND GENERALLY ENGAGED EMBRACED IN DOING SO. FINALLY THAT NCI THAT CATEGORY IT'S VERY IMPORTANT IN OUR VIEW FUNDING AGENCIES SUCH ADS THE NIH COLLABORATE AND TRANSLATIONAL SCIENCE INITIATIVES AND EFFECTIVELY ENGAGE OTHER FEDERAL AGENCIES, INDUSTRY, ACADEMIA, AND PHILANTHROPIC ORGANIZATIONS AS PARTNERS. I WILL ADD THE NOTE THAT WE BELIEVE IT'S EXCEEDINGLY IMPORTANT THE NIH NOT ONLY COLLABORATE WITH EXTERNAL PARTNERS IN THIS WAY BUT MORE FULLY COLLABORATE INTERNAL LATE THE NIH WITH OTHER ICs AMONG ICs IN THE TRANSINSTITUTIONAL WAY. IF YOU JUST LOOK AT THE BARE FACTS, LOOK AT THE NUMBERS. THE NCATS BUDGET 653 MILLION OR THEREABOUTS, FOR 2014. THE LARGEs SINGLE PROGRAM BUT LOOK AT THAT TIME OTHER INSTITUTES CLINICAL BUDGETS, THEY DWARF THAT 653 MILLION DON'T THEY? SO HOW CAN WE EXPECT NCATS COLLEAGUES TO CATALYZE TRANSFORMATIVE TRANSLATIONAL SCIENCE IN THE 62 CTSA SITES? IF THERE'S NOT FULLER COLLABORATION WITH THOSE OTHER IC TRANSLATIONAL AND CLINICAL PROGRAMS. I THINK -- WONDERFUL WE HAVE PETRA KAUFFMAN HERE WITH NCATS LEADING THE GENERAL CHARGE BECAUSE SHE WAS PLEA AGREEMENT PLAYER IN HER SERVICE AT NINDS IN FORMING NEURONEXT, A VIABLE CLINICAL NETWORK IN WHICH THE SO PETRA, WOULD YOU LIKE TO -- >> SURE. I'M IN A GOD POSITION BECAUSE UNTIL COUPLE OF WEEKS AGO I HELD INSTITUTIONS AT CATEGORICAL INSTITUTE WE HOPE ENGAGE WITH AND APPROACH TO MENTION -- CO-CHAIR A COMMITTEE THAT WAS FINDING OUT FROM THE CATEGORICAL ICs WHAT THEY WERE LOOKING FOR IN TERMS OF COLLABORATING WITH THE CTSA, WE FOUND A WIDE RANGE OF EXPECTATIONS AND SOME UNCERTAINTY ON HOW TO ENGAGE AS NETWORK, MANY MANY APER ABLE TO TAP INTO CTSA RESOURCES LOCALLY FOR DIFFERENT PROJECTS BUT LOOKING FOR A BROADER WAY TO COLLABORATE, SO ONE ISSUE WE TACKLE EARLY ON IS TO GO TOWER NIH COLLEAGUES TO PEOPLE WHO HOLD JOBS LIKE I HELD RECENTLY ACTUALLY -- THE RUBBER HITS THE ROAD. SPECIFIC OPPORTUNITIES TO ENGAGE AND ALSO TO LEADERSHIP TO TRY ON ALL LEVELS TO REALLY LEVERAGE THESE COLLABORATIONS AND WE COULDN'T AGREE MORE THIS IS REALLY A HUGE OPPORTUNITY FOR US. >> ONE COMMENT, GARY GIB BONNES WAS A MEMBER OF THIS COMMITTEE AND IT TAKES TWO TO TANGO AND COLLABORATE AND I KNEW GARY BEFORE SO I KNEW HIS THINKING AND HIS MO WHICH IS WHY I ASKED HIM TO BE ON THE COMMITTEE. HE IS A COLLABORATIVE PERSON. I WAS FRANKLY OVERWHELMED BY HIS ENTHUSIASM AND COMMITMENT FOR THIS TOPIC THAT PETRA AND RON ARE TALKING ABOUT. ENORMOUS EAGERNESS, NOT JUST WILLINGNESS BUT EAGERNESS TO ENGAGE THIS WAY SO IT'S REALLY AN ENORMOUS OPPORTUNITY. Q. I TO AD THAT WHEN GARY MADE HIS FIRST INTERVENTION ON THAT SCORE SITTING ACROSS THE TABLE FROM ME, TWO OF US GOT TO OUR FEET AND DID A HIGH FIVE. IT WAS JUST MANNA FROM BEETHOVEN HEAR GARY SPEAK TO NCI THOSE TERMS. WE HAD ALREADY HAD CONVERSATIONS WITH PETRA AND RAJESH TWO NINDS CLINICAL AND TRANSLATIONAL PEOPLE AND THEY SAID WOW, LET'S GET GOING NOW SO TREMENDOUS ENTHUSIASM THROUGHOUT THE NIH FOR COLLABORATIVE APPROACH ON TRANSLATIONAL SCIENCE IS JUST WE ARE THRILLED THAT'S THE AND THIS WORKING REPORT MIGHT BE ONE OF THE NECESSARY OR UNNECESSARY CATALYSTS TO GET THAT MOVING. SFROM SO THANK YOU, PETRA FOR THAT INTERVENTION. LET'S GO TO, AGAIN, GLASS HALF FULL HALF EMPTY, THESE ARE THE POSITIVE AND NEGATIVE FACTORS WE CONSIDERED IN THIS SPACE, THESE MERIT ALMOST FULL TREATMENT. WE SEE ON THE POSITIVE SIDE SCIENTISTS ARE ALREADY FORMING RESEARCH TEAMS WITH COLLABORATION AND OTHER PARTNERS. THERE ARE EXAMPLES OF ACADEMIC INSTITUTIONS DEVELOPING CRITERIA TO REWARD COLLABORATION. DIFFERENCE APPROACHES TO COLLABORATION INDUSTRY AND ACADEMIA BEING DEVELOPED. THE CTSA PROGRAM HAS ATTEMPTED IN A WAY TO ENGAGE THE PROCESS OF TRANSLATIONAL SCIENCES. ON THE OTHER HAND WE NEED TO GO FURTHER. OBVIOUSLY AS WE TALK ABOUT THERE'S DRAMATIC GROWTH IN PATIENT ADVOCATES AND COMMUNITY MEMBER WHOSE ARE EAGER TO PARTNER. NEGATIVE SIDE MANY ARE TANGENTIAL THAN TEN CENTRAL TO INTEREST OF THE COMMUNITY. THE CURRENT STRUCTURE SNOT BUILT FOR SCIENTIFIC ENGAGEMENT, GRANT INITIATED RESEARCH, ALL ABOUT GRANTS AND PUBLICATIONS THE GRANT RECIPIENT CALL IT IS SHOTS. AND THE COMMUNITY DOESN'T GET INVOLVED ESPECIALLY AT THE OUTSET OF DETERMINING WHAT PROJECTS SHOULD BE ADVANCED. THE REWARD SYSTEM IN GENERAL IS NOT ALIGNED WITH TRANSLATION HAL SCIENCE BUT MOST PART WHETHER MONEY PROMOTION ORISONS BELONGING OR RESPECT. WE ENCOURAGE COMPETITION RATHER THAN COLLABORATION IN THE CURRENT CULTURE AND WE NEED TO DO OTHERWISE. WE NEED TO BECOME -- TRANSLATIONAL SCIENCE IS A TEAM SUPPORT NOT AN INDIVIDUAL SPORT AND REWARD SYSTEM NEEDS TO RECOGNIZE THAT. COMMUNITY ENGAGEMENT IS LIMITED TO OUTREACH WHEN IT COMES TO RECRUITING MINORITY POPULATIONS AND WE NOW INADEQUATE THAT IS. I'LL READ THE MAIN BET BULLETS ON MEASURABLE OBJECTIVES WE CAME UP WITH, HOPE IN THIS THESE WILL BE HELPFUL IN TERMING SUCCESS AS WE GO ALONG. METH METHODS AN SYSTEMS ARE USED TO IDENTIFY RELEVANT AND DIVERSE STAKEHOLDERS ACROSS THE TRANSLATION SPECTRUM. SECOND, METHODS PROCESSES AND SYSTEMS FOR COLLABORATING ARE INNOVATIVE AND EVOLVE TO MEET THE NEEDS OF THE TRANSLATIONAL SCIENCE FIELD. THIRD TRANSLATIONAL SCIENCE COLLABORATIONS INVOLVE INDIVIDUAL, INDIVIDUALS ORGANIZATIONS AND DISCIPLINES THAT ARE APPROPRIATE TO THIS STUDY AIMS. EACH PART WITHIN A DIVERSE TRANSLATIONAL SCIENCE TEAM AS CAPACITY AND INFRASTRUCTURE SUPPORT TO BE A COLLABORATOR AND TO LEAD COLLABORATIONS AS COMPLEX TRANSLATIONAL SCIENCE PROJECTS EVOLVE. FIFTH, ESTABLISH MEASURES ASSESSING AND IMPROVING COLLABORATIONS. THAT DIVERSE STAKEHOLDERS AND COMMUNITY MEMBERS PLAY A KEY ROLE THROUGHOUT THE INFRASTRUCTURE OF THE CTSA PROGRAM. BOTH LOCALLY AND NATIONALLY. AGAIN, THESE ENGAGEMENTS AND COLLABORATIONS CAN HELP THE INDIVIDUAL CTSA SITE AND CTSA PROGRAM ACROSS THE BOARD NATIONALLY. FINALLY THE TRANSLATIONAL SCIENCE WORK FORCE IS COMPETENT IN COMMUNITY ENGAGEMENT AND COLLABORATION. WITH THAT I WILL INTRODUCE OUR DEAR COLLEAGUE SCOTT WHO WILL TAKE YOU THROUGH THIRD AND FOURTH STRATEGIC ROLE. >> NOT SURE THOUSAND FOLLOW RON BUT I WILL DO MY BEST HERE. THE THIRD STRATEGIC GOAL THE WORKING GROUP DEFINED AS INTEGRATION. WHAT I WOULD LIKE TO DO IS TO PERHAPS FRAME THE STRATEGIC OBJECTIVE IN THE FOLLOWING CONTEXT. CONSIDER FOR A MINUTE ACUTE SLIM FOESITIC IT TEASE MOST COMMON PEDIATRIC BLOOD CANCER, 4,000 NEW PATIENTS DIAGNOSED EACH YEAR. IN THE 60s THE SURVIVAL RATE FOR ALL WAS WHAT, ERIC, LESS THAN 10%. TODAY IT'S UP OVER 90%. THESE ALL PATIENTS AGAIN ARE BECOMING TEENAGERS, THEY'RE BECOMING ADULTS. THEY'RE NOT QUITE MY AGE BUT IN ANY RATE WHAT WE SEE NOW HEALTHCARE PROFESSIONALS ARE SEEING THAT THERE ARE HEALTH ISSUES THESE ALL CANCER SURVIVORS ARE ENCOUNTERING LATER IN LINE. CARDIAC ISSUES, PULMONARY ISSUES, INCREASED RISK FOR CANCER. WHAT I WOULD LIKE YOU TO DO IS CONSIDER FOR A MINUTE THE CTSA PROGRAM IS THE DESTINATION FOR TACKLING HEALTH ISSUE ACE CROSS THE ENTIRE LIFE SPAN. THAT'S THE FOCUS OF THE GOAL. TRANSLATIONAL SCIENCE IS INTEGRATEDDED, INTEGRATE AID CROSS THE MULTIPLE PHASES OF TRANSLATIONAL RESEARCH AND ACROSS THE DISCIPLINES THAT NEED TO WEIGH IN TO TACKLE COMPLEX PROBLEMS IN COMPLEX POPULATIONS ACROSS THE ENTIRE LIFE SPAN SO CONSIDERATIONS THAT THE WORKING GROUP IDENTIFIED WHERE ARE TO ACHIEVEST HEALTH IMPACT WITH GREATEST EFFICIENT, WE NEED TO FOCUS ON SCIENCE THAT WORKS. TRANSLATIONAL SCIENCE WILL CAPTURE THE CRITICAL RELATIONSHIPS THAT IN SOME CASES ARE FRAGMENT RECORD PERHAPS MORE NARROW APPROACHES WILL AND CURRENTLY ARE MISSING. TRANSLATIONAL SCIENCE WILL PLAY A PIVOTAL ROLE IN UNCOVERING THE RELATIONSHIPS BETWEEN DISEASE, ENVIRONMENTAL EXPOSURES, HEALTH RELATED BEHAVIORS AND THE SOCIETAL FACTORS ACROSS THE LIFE SPAN AND TRANSLATIONAL SCIENCE IS CRITICAL TO UNDERSTANDING OF THE CAUSES OF HEALTH DISPARITIES AND HOW THEY RELATE TO SOCIAL ECONOMIC HEALTH SYSTEMS FACTORS. WE DEFINE WHAT WOULD SUCCESS LOOK LIKE IF THE CTSA PROGRAM WAS COMPLETELY INTEGRATED IN THIS REGARD? TRANSLATIONAL SCIENCE INCLUDES EFFORTS TO STUDY SPECIAL POPULATIONS AND DIFFERENCES IN THE PROGRESS OF DISEASE, THE TREATMENT OF DISEASE PROCESSES, WOULD THEN BE DEFINED THROUGH ACHIEVING THE STRATEGIC GOAL. THE EFFORTS LEAD THE QUANTIFIABLE IMPROVEMENTS TO HEALTHCARE, HEALTHCARE OUTCOMES AND QUALITY OF LIFE FOR PEOPLE WITH CHRONIC DISEASES AND AS WELL FOR RACIAL ETHNIC AND UNDERSERVED POPULATIONS. LABORATORY AND CLINICAL ADVANCES WILL BE TRANSLATED RAPIDLY INTO LIFE SAVING AND LIFE PROLONGING INTERVENTIONS. AND NEW MODELS WILL BE CREATEDDED THAT WILL INCLUDE REGULATORY, ETHICAL AND POTENTIALLY POLICY CONSIDERATIONS. THAT INCLUDE ALL PATIENTS IN BIOMEDICAL RESEARCH. THERE WILL BE OPPORTUNITIES TO PREVENT, POSTPONE THE ONSET, OR OTHERWISE ALTER THE NATURAL HISTORY OF ACUTE AND CHRONIC DISEASES, DATA ACROSS THE LIFE SPAN IS INTEGRATEDDED ANALYZED, DISPLAYED AN EXPLOITED AS RELATED TO TRANSLATIONAL SCIENCE. THEN LASTLY, TRANSLATIONAL INVESTIGATION OF HEALTH CONDITIONS THAT ARE SPECIFIC TO THE DIFFERENT LIFE STAGES ARE EMPHASIZED. WE IDENTIFY POSITIVE FACTORS, WHAT ARE NEGATIVE FACTORS THAT IMPACT SUCCESS. IN THIS REGARD. THE C THETSA PROGRAM AND CENTERS WE FEEL ARE UNIQUELY POSITIONED TO PLAY A COORDINATING OR CONVENING ROLE IN BRINGING TOGETHER SCIENTISTS. FROM DISPARATE FIELDS TO STUDY DISEASES LONGITUDINALLY. I LIKE TO BRING YOU BACK TO THE ALL EXAMPLE. THERE ARE BARRIERS THOUGH. THERE ARE BARRIERS TO CONDUCTING TRANSLATIONAL RESEARCH ACROSS THE LIFE SPAN. WE HAVE SCIENTISTS FOCUSED ON SPECIFIC FIELDS THAT DON'T INTERACT WITH COLLEAGUES IN OTHER FIELDS. PATIENTS ARE NOT ENGAGED AS COLLABORATIVE PARTNERS IN THE CLINICAL CARE AND RESEARCH PROCESS AND AS I HOPE TO HIGHLIGHT IN THELL EXAMPLE A RESEARCH GAP IN THE TRANSITION PHASES. OF LIFE SO WHAT WILL IT TAKE TO GET THERE? TRANSLATIONAL SCIENCE ADDRESSES SPECIAL POPULATIONS. CHILDREN, ELDERLY, LATINOS, AFTER CAN AMERICANS AND ALL TRANSITIONS ACROSS THE LIFE SPAN. SECONDLY TRANSLATIONAL SCIENCE ADDRESSES ALL TYPES OF INTERVENTIONS. WHETHER THAT'S A DRUG, A MEDICAL DEVICE, A DIAGNOSTIC BEHAVIORAL CHANGE IN ALL POPULATIONS, TRANSLATIONAL SCIENCE FROM INTEGRATED TO CARE RESEARCH CARE POPULATIONS ACROSS THE SPECTRUM OF THE LIFE SPAN. FOURTH, TRANSLATIONAL RESEARCHERS INTEGRATE THE ENTIRE TRANSLATIONAL SCIENCE SPECTRUM. HOW THEY CONCEPT ACTUALIZE AND IMPLEMENT RESEARCH FOR PATIENTS THROUGHOUT THE LIFE SPAN. AND FIFTH, THE METHODS THAT INFLUENCE INTEGRATION OF EVIDENCE BASED INTERVENTIONS INTO PRACTICE SETTINGS BECOME FULLY REALIZED. FOURTH GOAL FOURTH AND FINAL GOAL THAT WE DESCRIBED IN OUR REPORT, PROVIDED TO NCATS IS AROUND METHODS AND PROCESSES. THERE THE SCIENTIFIC STUDY IN THE PROCESS OF CONDUCTING TRANSLATIONAL SCIENCE ITSELF ENABLES SIGNIFICANT ADVANCES IN TRANSLATION T. CONSIDERATION, RAPID PACE OF TRANSFORMING DISCOVERIES ARE TRANSPERFORMING OUR WORLD AND WITH THESE EMERGENT RELATIVELY UNCHARTERED TERRITORIES, SCIENTISTS MUST DEVELOP EVERY STEP OF THEC' PROCESS THAT ITSELF IS COMPLEX, DYNAMIC AND FREQUENTLY NON-LINEAR. SO WE REALLY HAVE TO REIMAGINE THE TRANSLATIONAL SCIENCE PROCESS. WE VALUE IT HERE TO HIGH QUALITY METHODS AND PROCESSES AND DEVELOP NEW TECHNOLOGIES NEW APPROACHES METHODS AND P POLICYINGS TO SPEED TANGIBLE IMPROVEMENTS TO HUMAN HEALTH. WHAT DOES SUCCESS LOOK LIKE? THE PROGRAM FUNCTIONS INDIVIDUALLY AND AS A RESEARCH ENGINE. THEY ARE ROUTINELY ESTABLISHED IN NEW SCIENTIFIC FIELDS AND PARADIGMS AND DEVELOP TECHNOLOGIES AND METHODS THAT CHANGE THE WAY SCIENTISTS APPROACH THEIR WORK. BY CHANGING THE METHODS AND PROCESSES THE TRANSLATIONAL SCIENCE PROCESS DRAMATICALLY IMPROVES AND ACCELERATING TRANSLATION OF DISCOVERIES INTO PRACTICE IN REAL WORLD SETTINGS. ROADBLOCKS WE FACE ARE IMPEDE THE PROCESS TODAY ARE IMPROVED AND IMPACT HUMAN HEALTH. NEW TOOLS TECHNOLOGIES DATA SETS MODELS ARE WIDELY AVAILABLE TO ENABLE TRANSLATION ACROSS ORGANIZATIONS ACCELERATE TRANSLATION OF SCIENCE AND TEST APPROACHES FOSTER EVENVATION IN REAL WORLD SETTINGS DATA SETS ARE INTEGRATED DATA SHARING AND ACCESS TO SECONDARY DATA IS THE NORM. SO SEVERAL POSITIVE AND NEGATIVE FACTORS THE STANDARDIZATION OF METHODS AND PROCESSES IS AN OPPORTUNITY TO FREE INVESTIGATORS TO REALLY THEN FOCUS ON INNOVATION. THE CTSAs REALLY HAVE ACCESS TO DIVERSE EXPERTISE THAT'S NEEDED TO REDESIGN TRANSLATIONAL SCIENCE PROCESS. THE CTSAs NOW HAVE THE FLEXIBILITY TO FOCUS ON NEW AREAS OF SCIENCE. ON THE DOWN SIDE THERE ARE NO CURRENTLY NO ESTABLISHED STANDARDS IN TRANSLATIONAL SCIENCE FROM PERFORMANCE MEASURES. NO CARD HARMONIZATION AMONG CTSA SITES, THE REGULATIVE SCIENCE COMPETENCIES OF RESEARCHERS WITHIN OUR CONSORTIUM ARE FRANKLY NOT STRONG. THE CTSA HISTORICALLY HAVE NOT HAD A STRONG FOCUS ON THE SCIENCE OF TRANSLATIONAL SCIENCE. SO WHAT WILL IT TAKE TO GET THERE? WE DEVELOP METHODS AND PROCESSES THAT OPTIMIZE PROCESSES TO CARRY OUT TRANSLATIONAL SCIENCE WITHIN DIFFERENT STAKEHOLDER ENVIRONMENTS. WE EMERGING AREAS IN SCIENCE THAT PROCEED MORE QUICKLY DUE TO CTSA DRIVEN INNOVATIONS. THE BUSINESS OF TRANSLATIONAL SCIENCE IS MANAGED WITH THE ACCOUNTABILITY AND DATA DRIVEN DECISION MAKING. THE METHODS AND PROCESSES ARE OPTIMIZED FOR REGULATED AND NON-REGULATED INTERVENTIONS, SO THAT REQUIREMENTS ARE TRANSPARENT, AND THEY'RE CONSISTENTLY APPLIED. WE HAVE AN LIT ACTION COMPUTATIONAL TOOLS TO MANAGE EXPLOSION OF DATA AND ENABLE OPTIMAL UTILIZATION OF THOSE DATA TO MAKE TANGIBLE IMPROVEMENTS IN HUMAN HEALTH. THAT THE TANGIBLE IMPROVEMENTS ARE MADE IN A TIMELY FASHION. AND QUALITY MANNER AND ETHICAL STANDARDS ROVE SEARCH ARE APPLIED. AS EXAMPLE, HUMAN SUBJECTS PROTECTIONS, IRBs, HERE WHAT WE TALKED ABOUT WITHIN THE WORKING GROUP IS IMAGINE FOR A MINUTE THERE'S BEEN A DISCOVERY THAT'S A PROMISING NEW TREATMENT FOR A RARE DISEASE. A DISEASE FOR WHICH CURRENTLY THERE ARE NO TREATMENTS AND IMAGINE FOR A MINUTE AN INNOVATOR COULD GO TO CTSA PROGRAM, THE CTSA PROGRAM COULD BE THE DESTINATION, FOR CLINICAL PROOF OF CONCEPT FOR THAT EXCITING POTENTIAL PROMISING NEW TREATMENT. AND TRIAL ESTABLISHED AT 60 SITES WITHIN 30 DAYS AND ENROLLMENT FLEET COMPLETED WITHIN 12 MONTHS. SO TO WRAP UP, I THINK ON BEHALF OF THE WORKING GROUP WE URGE THE COUNCIL MEMBERS TO DO IS WE APPRECIATE YOU REVIEWING THE REPORT AND PROVIDING FEEDBACK. JUST TO CONCLUDE WHAT WE'RE HOPING TODAY, NORA AND RON AND I, TO PROVIDE HIGHLIGHTS CONTAINED WITHIN THE WORKING GROUP REPORT. 6 AND WHAT YOU'LL SEE IN THE REPORT IS SPEEDING UP THE PROCESS FOR TRANSFORMING DOUSE DISCOVERY TO APPLICATION, IS CLEAR LAY TOP PRIORITY FOR NCATS. REDESIGNING AN ACCELERATING THE PROCESS OF TRANSLATIONAL SCIENCE ENABLE NEW DISCOVERIES TO REACH PATIENTS AND COMMUNITIES FASTER. THE WORKING GROUP IDENTIFIED CRITICAL ISSUES AND OPPORTUNITIES ACROSS THE ENTIRE TRANSLATIONAL SCIENCE SPECTRUM FOLLOWED BY DEVELOPMENT OF MEASURABLE GOALS AND OUTCOMES FOR THE PROGRAM. AND THAT WE ADDRESSED FOUR OF THE SEVEN RECOMMENDATIONS OUTLINED IN THE IOM REPORT. MEASURABLE GOALS AND OUTCOMES DEVELOPED BY THE WORKING GROUP WE HOPE WILL SERVE AS A GUIDE FOR MEASURING AND REPORTING PROGRESS ON THIS CRITICALLY IMPORTANT PROGRAM. THANK YOU. [APPLAUSE] >> WOW. THANK YOU. TO THE COMMITTEE, I WAS MADLY TAKING NOTES HERE. FIRST I WANT TO THANK YOU FOR THE INCREDIBLE WORK YOU DID. I LOST TRACK OF THE FACT YOU DID ALL YOUR WORK IN FIVE MONTHS WHICH WAS INCREDIBLE. I'M REALLY EXCITED TO HEAR WHAT Y'ALL CAME UP WITH. I THINK IT REALLY GIVES US SOME REALLY TANGIBLE OUTCOMES THAT WE CAN AIM FOR. OUR PLAN WITH NCATS IS GOING TO BE DEVELOPED IMPLEMENTATION OF THESE RECOMMENDATIONS WITHIN THE PROGRAM AS RAPIDLY AS WE CAN. WE CERTAINLY GOING TO DO AS WE DO THAT TO KEEP IN THE TOUCH WITH ALL OF YOU AND CTSA COMMUNITY AT LARGE AS WE DO THAT, DURING THAT PROCESS THIS IS THE FIRST I HAVE SEEN OF THIS, I'M SORT OF RESPONDING TO IT TOO BUT I'M GOING TO HAVE LOTS OF TIME TO THINK ABOUT THIS. SO WHAT I WOULD LIKE TO DO IS TAKE ADVANTAGE OF ALL INTELLIGENCE IN THE ROOM TO GET YOUR IMPRESSIONS ABOUT WHICH OF THESE PRIORITIES WE OUGHT TO FOCUS ON, IF THERE ARE ONES YOU THINK THEY MISSED. WE WANT TO HEAR THAT. IF THERE ARE OTHERS YOU THINK ARE OFF BASE WE WANT TO HEAR THAT TOO. THIS IS A WORKING GROUP THAT'S ADVISORY TO THE COUNCIL. SO THIS IS REPORT TO COUNCIL, NOT JUST TO ME. I WANT TO SAY BEFORE WE DO THAT THAT EMAIL ADDRESS RIGHT THERE IS HOPE YOU WILL NOT ONLY NOW BUT IN THE DAY AS YOU TRAVEL BACK TO YOUR HOMES PERHAPS ON THE PLAIN PLANE OR WHAT HAVE YOU TO SEND US COMMENTS IDEAS AND THOSE OF YOU WHO ARE OUT IN THE ETHER WERE VERY ANXIOUS TO HEAR EAGER TO HEAR YOUR COMMENTS AS WELL SO I'M SPEAKING TO THE COLLECTIVE UNCONSCIOUS HERE THAT I CAN'T SEE, SO THOSE WHO ARE ARE WATCHING ON VIDEO, I HOPE YOU WILL SEND COMMENTS AND THOUGHTS AS WELL. THE REPORT HAS BEEN POSTED TO OUR WEBSITE CONCURRENT WITH THE START OF THIS PRESENTATION SO THOSE CONNECTED TO THE WEB SHOULD BE ABLE TO GET IT NOW. REWE QUEST YOU AND HERE AT HOME TO GET COMMENTS TO US IN TWO WEEKS BECAUSE WE WANT TO MOVE FORWARD AS RAPIDLY AS POSSIBLE NOW. WITH THAT I WOULD LIKE TO OPEN THE FLOOR TO COMMENTS AND QUESTIONS, ET CETERA. >> I CERTAINLY DON'T HAVE SUGGESTIONS WHAT'S MORE OR LESS IMPORTANT, IT DOES COME TOGETHER. I GUESS THE COMMENT I WANT TO MAKE AND YOU HEAR AND OTHER SUBCOMMITTEE REPORTS IS THAT I REALLY SUPPORT THOUGHTFUL DATABASED MANNER IN WHICH THESE WERE MADE. YOU'LL HEAR FROM US LOOKING AT SYSTEMS, MEASURE, HAVING MEASURABLE OBJECTIVES KNOWING WHAT IT IS YOU WANT TO ACCOMPLISH BEFORE YOU BEGIN, SO THAT BACKING OFF AND LOOKING AT SAYING WE WANT TO DO SOMETHING BUT WHY DO WE WANT TO DO IT AND STARTING WITH WHAT IS IT YOU WANT TO ACCOMPLISH AND LOOKING HOW TO DO IT AND KNOWING AHEAD OF TIME WHAT MEASURABLE OBJECTIVES DEFINE SUCCESS, I WANT TO COMMEND THAT. >> I THINK YOU GUYS HIT A HOME RUN. CONGRATULATIONS. THIS IS TERRIFIC. TERRIFIC WORK. THE TWO POINTS THAT I WANT TO PICK UP ON ARE NOT NECESSARILY RELATED TO ONE ANOTHER. IT'S INTERESTING TO ME THAT THE IOM REPORT SORT OF CALLS OUT PEDIATRICS AND I THINK YOU DID AN EFFECTIVE JOB TRANSFORMING THAT, AS A PEDIATRICIAN I HAVE A CONFLICT OF INTEREST HERE AND SOME SENSE WOULD LIKE TO HAVE SEEN MORE CHILD ADVOCACY PUSH. THAT IS NOT NCATS JOB, THAT MAYBE SOMEWHERE TO WORK WITHIN NICHD. BUT THE AOL EXAMPLE IS BEAUTIFUL BECAUSE IT'S NOT EVEN A CANCER PROBLEM ANY MORE. THESE SURVIVORS HAVE ENDOCRINE PROBLEMS AND THEY HAVE PULMONARY PROBLEMS. THEY HAVE BONE PROBLEMS, YOU NAME IT. SO IT IS A REALLY NICE EXAMPLE AND WITH WORK ON TAKE TAG IOM AND MAKING IT NCATS I THINK IS GREAT. THE SECOND AREA AREA THAT I THINK IS QUITE INTERESTING, I WOULD BE'S TORE HEAR WHAT OTHER MEMBERS OF COUNCIL THINK ABOUT THIS, THE IRB RELIANCE, THE PIECE OF THE NCATS WEBSITE ABOUT THIS MOVE TO REALLY -- WE DON'T HAVE POWER AND CONTROL OVER THIS BUT WE CAN HAVE INFLUENCE TO REFRAME HOW IRBs FUNCTION IN THE 21st CENTURY FROM AN APPROACH THAT SAYS NORTH STAR IS PROTECTING PEOPLE FROM RESEARCH RISK TO POTENTIALLY A SYSTEM THAT'S -- THERE ARE BALANCE ISSUES. AND ACCELERATING PROCESS FOR SICK PEOPLE AND FOR ALL PEOPLE IS AN EQUALLY IMPORTANT GOAL. TO PROTECTING FROM RESEARCH RISK AND I THINK THIS REPORT HELPS THAT. >> I WANT TO ADD A NOTE OF CONGRATULATIONS, I REALLY RESPECT WHAT YOU HAVE DONE WITH MEASURABILITY OF THIS. ONE QUESTION IN OUR WORK ON TECHNOLOGIES, WE ARE NOTICING WITH STUDENTS WE WORK ON MORE GLOBAL AS IN OUTSIDE THE U.S. CONNECTION. I DIDN'T SEE THAT PROMINENTLY IN INTEGRATION OR COLLABORATION, I WONDER IFLŽ—U DISCUSSED THAT AS A THEME. >> NOT AS A MAJOR THEME, IT WAS A SUBTHEME IN TERMS OF COLLABORATION AND IF YOU LOOK AT THE REPORT THE FULL REPORT WE HAVE STATEMENTS ABOUT THIS NEEDS TO GO WORLDWIDE TAKING A WORLDWIDE VIEWPOINT ESPECIALLY IN TERMS OF EDUCATING THE WORK FORCE. AND THINGS ON THOSE LINES. YOU'LL FIND IT AS A FLAVOR IN THE REPORT ITSELF BUT AS SOMETHING CALLED OUT AS AN INDIVIDUAL WE DID NOT. THOUGH IT IS IMPLIED IN MANY WAYS IN THE REPORT YOU DO COLLABORATE AND ALIGN WITH THE FDA. I DON'T SEE IT STATEDDED EXPLICITLY AND LIKE TO ENCOURAGE FOR THE FUTURE FOR A NUMBER OF REASONS NOT JUST POLITICAL, I WOULD LIKE TO COMMENT ALL OF YOU WHO WORKED IN FOR EXAMPLE CORE COMPETENCIES PROGRAM AND THE ALIGNMENT OF THAT WITH REGULATORY SCIENCE AT FDA. THERE'S BEEN SOME GOOD WORK DONE. AND PROJECTS CONDUCTED IN COLLABORATION AND OTHER EXAMPLES. WE NEED TO MAKE THIS MORE PROMINENT THIS PROGRAM IS ALIGNED THE FDA SCIENCE PROGRAM. >> IN THE ABSENSE OF SEEING THE FULL REPORT, I WAS WONDERING HOW THE COMMITTEE ADDRESSED THE ONE OF THE PRIMARY CONCERNS RAISED IN THE IOM REPORT WAS THE EVALUATION PROCESS FOR THE CTSAs. AND THEIR RECOMMENDATION WAS TO STANDARDIZE AND FORMALIZE THAT PROCESS. WHILE I SEE THE ELEMENTS OF MEASUREMENT IN THIS EXTENSIVE AND COMPLETE REPORT THAT YOU PROVIDED, I WASN'T QUITE SURE HOW THESE ELEMENTS GOT PUT INTO AN EVALUATION PROCESS. >> I'LL LET YOU TAKE THAT CHRIS BECAUSE THE IMPLEMENTATION AND THE DEVELOPMENT OF METRICS, THIS IS THE TEMPLATE THAT NCATS WILL USE, THAT WAS IN PART OF OUR SPECIFIC SCOPE OF WORK. >> I SEE THIS A TRANSLATIONAL STEP FROM THE IOM REPORT TO WHAT WE NOW HAVE TO DO, WHAT WAS EVIDENT WHEN THE IOM REPORT CAME OUT WAS THEY DID SAY THAT MEASURABLE OBJECTIVES ARE CRITICAL, EVALUATION IS CRITICAL AND WE'RE BIG ON THAT. IF YOU GO BACK TO THE 3-Ds IF YOU DEMONSTRATE SOMETHING, HOW DO YOU KNOW IF YOU DEMONSTRATE THAT OR NOT? IT CAN'T JUST BE NUMBERSES OR TRANSACTIONS WHICH WE HAVE HAD IN THE PAST. WHAT WE'RE ENDEAVORING TO DO IS TO TAKE THESE RECOMMENDATIONS OF THE DIRECTIONS WE SHOULD GO IN AND THEN DIFFICULTY WAS GOING TO BE OR THE CHALLENGE FOR US IS GOING TO BE CREATE A FUNDING PROGRAM RFA, THAT HAVE WITHIN THEM MEASURABLE OBJECTIVES AND OUTCOMES THAT ARE PART OF PART AND PARCEL AND PART OF THE CULTURE OF THE PROGRAM GOING FORWARD. AS YOU PROBABLY ALL KNOW, IN A VERY WEEDY LEVEL ONE CAN AND OFTEN DOES AND SHOULD INCORPORATE MEASURABLE OBJECTIVES IN NOTICES OF GRANT AWARD AND THESE THINGS GOING FORWARD. WE DIDN'T WANT THIS REPORT, THIS GROUP TO DO THAT BECAUSE THAT'S REALLY A PROGRAM RESPONSIBILITY TO DO. THE OTHER THING WE ASKED THEM TO DO LOOK FORWARD TO SEEING IN THE REPORT WAS TO FIND OUT WHETHER WE'RE HOW DO WE KNOW WE'RE MEASURING THE RIGHT THING? THAT'S THE THING WE'RE AFRAID OF, THERE'S LOTS OF EFFORTS TO MEASURE THINGS IN THE PAST AND OF COURSE YOU GET WHAT YOURSELF YOU MEASURE BUT OFTEN WHAT YOU MEASURE INDUCES ALL KINDS OF UNINTENDED CONSEQUENCES, SO IT IS NOT ONLY WHAT ARE WE GOING TO MEASURE BUT WHAT ARE WE GOING TO MEASURE THE RIGHT THING TO MEASURE. I THINK I SAID THAT RIGHT. FIRST DERIVATIVE OF MEASUREMENT BASICALLY. THIS IS WHAT WE HAVE TO DO BECAUSE OUR TASK AND NOT TO PUT TOO FINE A POINT, PETRA'S TASK IS TO NOW WRITE THE RFAs WHICH HAVE TO INSTANTIATE WHAT YOU'RE TALKING ABOUT. AND LIKE IN EVERYTHING WITH NCATS IS GOING TO BE EXPERIMENT, I DON'T PRETEND THAT WE'RE GOING TO GET MEASURABLE OBJECTIVES RIGHT THE FIRST TIME. SO WHAT WE'RE GOING TO DO IN WRITING THE IS TO GATHER DATA ON WHETHER WE'RE NOT ONLY WHAT THE PRODUCTS IF YOU WANT TO THINK THAT WAY OF THE PROGRAM ARE, BUT BY PUTTING MEASUREMENTS IN PLACE, WE HAVE SKEWED THE PROGRAM IN A POSITIVE DIRECTION OR A NEGATIVE DIRECTION. IT'S ALL ABOUT INCENTIVES, WE WANT TO PUT INCENTIVES IN THE RIGHT PLACE. THAT'S AS YOU KNOW, THAT'S A FIELD IN ITS OWN RIGHT. I NOTICED WITHIN THIS MULTIPLE THINGS, THAT I ACTUALLY CIRCLED WHERE THE COMMITTEE TALKED ABOUT IMPORTANCE OF A SCIENTIFIC APPROACH AND EXPERIMENTAL APPROACH SCIENCE OF MEASUREMENT. WHICH IS A FIELD INTO ITSELF AS YOU ALL KNOW. BUT ANY SUGGESTIONS HOW TO DO THIS WOULD BE GREATLY ENCOURAGED. >> CHRIS, ASK YOU A QUESTION. DO YOU HAVE THE DISCRETION WITHIN THE CTSA BUDGET TO PULL OUT DOLLARS? THIS REQUIREMENT YOU HAVE HERE THIS WILL REQUIRE BIG INVESTMENT IN INFRASTRUCTURE AND SYSTEMS, IF YOU'RE GOING TO STRIVE TO PULL TOGETHER CTSAs TO DO THE CLINICAL TRIALS YOU SPOKE ABOUT YES THERE IS A FORMULATION OUT THERE, GRANTS, THESE INSTITUTIONS EXPECT GRANTS TO HAVE GREAT EXPECTATION OF THIS POT OF MONEY BUT THERE NEEDS TO BE AN INVESTMENT IN INFRASTRUCTURE TO TIE TOGETHER THESE CENTERS AND THE KINDS OF PROGRAMS THAT ARE NEEDED HERE AND HAVE BEEN DESCRIBED AND SHOULD BE MEASUREDDED BECAUSE YOU CAN'T MEASURE IT UNLESS YOU HAVE THAT. DO YOU HAVE THE DISCRETION TO DO THIS IN THE PROCESS WITH CURRENT CTSA BUDGET? I SHOULDN'T USE THE TERM SLUSH FUND BUT IS THERE A FUND AVAILABLE FOR THAT AND CAN YOU TAKE IT FROM THERE IS THERE SOMETHING TO GIVE YOU THAT OPPORTUNITY? WE HAVE INSTITUTIONS OUT THERE EXPECTING X MILLION DOLLARS AND WE START PULLING FROM THAT TO SUPPORT THIS, I'M WORRIED -- I THINK THIS IS GREAT. IT COMES DOWN TO THE IMPLEMENTATION AND THE DISCRETION THAT YOU HAVE TO PUT THESE THINGS TO REALLY THIS REPORT DOESN'T GIVE YOU THAT DISCRETION, YOU WERE CAREFUL AND I APPROPRIATELY SO TO SEPARATE IMPLEMENTATION TO -- THAT'S THE AGENCY RESPONSEN'T, OURS IS IS THE OTHER BUT QUITE FRANKLY ARE WE MISSING AN OPPORTUNITY TO GIVE YOU DISCRETION TO DO THAT SOMEWHERE -- AND HOW ARE YOU GOING TO GET THAT? >> GREAT QUESTION, THANK YOU FOR ASKING. WE'RE ALL AWARE THIS IS A PROGRAM THAT NEEDS TO EVOLVE TO MEET NCATS MISSION. THERE ARE REALLY REMARKABLE SCIENTIFIC OPPORTUNITIES THAT WERE NOT AROUND WHEN THIS PROGRAM STARTED MUCH LESS PREDECESSORS. THERE IS DISCRETION WITHIN THE PROGRAM THE TENSION THAT WE'RE WORKING ON IS HOW TO EVOLVE THE PROGRAM AS RAPIDLY AS POSSIBLE UTILIZING THAT DISCRETION WHILE AT THE SAME TIME REALIZING THE PREVIOUS MODEL CREATED ITSELF AN ECOSYSTEM WHICH IS SOMEWHAT DEPENDENT ON THAT PREVIOUS MODEL. SO WE NEED TO BE SENSITIVE TO THE FACT THIS HAS TO EVOLVE NOT BE EVOLUTION HAS TO BE GRADUATED INTERPUNCTION WALL EVOLUTIONARYISM. I THINK THE OTHER THING TO SAY IS THAT WHEN I LOOK AT THE REPORT OF THE RECOMMENDATIONS HERE, IF YOU ASK WHO WOULD DO THESE RECOMMENDATIONS? WHO ARE THE INSTITUTIONS THAT DO THIS WORK? ARE THEY DIFFERENT FROM THE INSTITUTIONS THAT CURRENTLY HAVE CTSAs? THE ANSWER IS CATEGORICAL TERMS, NO. THEIR ACADEMIC INSTITUTIONS SO OUR EXPECTATION WOULD BE THAT WHEREVER, HOWEVER WE GO FORWARD WITH IMPLEMENTING THESE RECOMMENDATIONS THE ORGANIZES, THE INSTITUTIONS THAT HAVE RELIED ON THESE RESOURCES AND DO RELY ON THESE RESOURCES WILL BE THOSE WHO WILL BE ELIGIBLE FOR THESE FUNDS TOO AND WE HOPE OTHERS THE LARGER ACADEMIC COMMUNITY. SO I THINK TO SAY AS I HAVE GOTTEN TO APPRECIATE WHAT THE CTSAs HAVE DONE, I WOULD BE INTERESTED WHAT NORA AND SCOTT AND OTHERS THINK WHO ASSOCIATED WITH THE CTSAs, INDIVIDUAL CTSAs HAVE WORKED ON VIRTUALLY ALL OF THESE PROBLEMS IN SMALL WAYS. SO DIFFERENT ONE VERSUS DIFFERENT STRENGTHS IN THESE AREAS. SO ONE OF THE CHALLENGES FOR US AND U YOU SAW IT IN RECOMMENDATION THREE OF THE IOM REPORT THESE FOLKS TALK ABOUT WE ASKED -- WE DIDN'T ASK TO TALK ABOUT WAS HOW DO YOU ALLOW INDIVIDUAL INSTITUTIONS TO PLAY ON THEIR STRENGTHS. THOSE COULD BE CURRENT CTSA INSTITUTIONS, ACADEMIC INSTITUTIONS, THAT HAD A CTSA ONCE AND DON'T HAVE ONE NOW, OR NEVER HAD ONE BUT MIGHT HAVE PARTICULAR EXPERTISE IN A TRANSLATIONAL AREA WHICH THE REPORT TALKS ABOUT SO THIS WILL ACTUALLY ALLOW US TO INCREASE THE SIZE OF THE TENT. SO I THINK THE ISSUE IS NOT SO MUCH AS FAR AS MY POINT OF VIEW SO MUCH DISRUPTION, I THINK WE HAVE THAT DISCRETION, THE ONE THING THAT HOW FUNDS ARE DISTRIBUTED, ONE THING WE DON'T HAVE WHICH IS A SEPARATE ISSUE, IS DISCRETION, STATUTORY LANGUAGE HOW MUCH MONEY NEEDS TO BE SPENT ON THIS PROGRAM EVERY YEAR. THAT'S IN OUR APPROPRIATION. WE HAVE TO SPENT A DOLLAR NUMBER ON THIS PROGRAM. BUT WITHIN THAT IT IS OUR RESPONSIBILITY AS PUBLIC SERVANTS AND AS PART OF THE AGENCY TO WITH YOUR HELP DECIDE HOW THE PUBLIC HEALTH CAN BENEFIT MOST FROM THESE MONEYS WE HAVE BEEN TOLD WE NEED TO SPEND. SO I GUESS WHAT I WOULD SAY IS THAT AS ANY CHANGE HUMAN BEINGS DON'T LIKE CHANGE. WE'RE ALL AWARE OF THIS. I THINK THERE ARE GOING TO BE -- JUST GOING TO BE SOME DISCOMFORT, ALREADY HAS BEEN, AS WE EVOLVE THIS PROGRAM. SO YOUR HELP AS THE COUNCIL IN EXPLAINING AND ADVISING US ON HOW TO DO THIS, IS REALLY IMPORTANT AS WE MAKE IT THROUGH THIS TRANSITION. I GUESS THE LAST THING TO SAY, WHEN I LOOK AT THE C THETSA PROGRAM I LOOK AT OPPORTUNITIES HERE. THE DIFFICULTY IS NOT HOW WE SPEND THE MONEY WE HAVE BEEN TOLD. THE QUESTION IS, HOW ON EARTH DO WE DO THIS MANY TRANSFORMATIONAL THINGS WITH A BUDGET THAT'S THAT SMALL. IT'S A LOT OF MONEY BUT GIVEN WHAT WE HAVE TO DO, IT'S NOT BIG ENOUGH. AND THE WAY TO DO THIS, RON MENTIONED THIS BEFORE, ONE REASON WHY WE'RE SO INTENT ON HAVING MORE ROBUST INTERACTIONS WITH THE INSTITUTES AND CENTERS AND MORE ROBUST INTERACTIONS ON THE CLINICAL SIDE WITH PHARMA, WITH BIOTECH, WITH OTHER PARTS OF THE RESEARCH ECOSYSTEM SO THAT SO THE PIE GROWS. AND IT REALLY CAN IF WE CAN DO IT FROM A TOTAL FUNDING PERSPECTIVE. >> JUST TO FOLLOW-UP ON DR. BELL 'S COMMENTS I THINK IF WE WERE TO SYNTHESIZE THAT DOWN (INAUDIBLE) IT'S FAIR TO SAY COUNCIL SUPPORT FOR NCATS HAVING THIS QUESTION WITHIN THE CTSA PROGRAM TO IMPLEMENT THE RECOMMENDATIONS, IS MOST WELCOME. >> THAT'S WHAT I WAS TRYING TO GET AT, I DON'T KNOW WE'RE PREPARED TO DO IT BECAUSE WE HAVEN'T SEEN THE REPORT. BUT MAYBE WHAT -- BECAUSE I WOULD LIKE TO SEE, PERSONALLY, I THINK THE ISSUE IS GOING TO BE MAKING SURE THERE IS A POT OF MONEY FOR DISCRETION FOR INN INVESTIGATION, FOR TYING THE THINGS TOGETHER. YET, I RECOGNIZE THE POSITION YOU'RE IN BECAUSE YOU HAVE EVERY CONGRESSMAN AND EVERYBODY LIKE THAT IF THEY SEE A 10% DROP IN CTSA THEY'RE BANGING ON YOUR DOOR. AND THIS I THINK THE REPORT COVERS IT. BUT I THINK UNLESS SOMEBODY OR SOME GROUP SAYS THERE NEEDS TO BE X NUMBER OF DOLLARS TO BE ABLE TO PULL FROM BECAUSE ADMIT IT, FOR A WHILE CTSA BUDGET IS NOT GOING TO GROW UNLESS YOU GO AROUND WITH YOUR TIN CUP FROM THE VARIOUS INSTITUTES P AND BIDs, ET CETERA, ET CETERA, IS NOT GOING TO GROW, THEY GOT THEIR OWN ISSUES. I THINK YOU GOT A POT OF MONEY, HOW ARE YOU GOING TO USE THE POT OF MONEY WITH DECISIONS MADE EVERY DAY. WE SET PRIORITIES, EVERYBODY HAS A PRIORITY. THERE'S SOME WAY WE CAN HELP IN TRYING TO UNDERSCORE THAT BECAUSE I DON'T THINK YOU'RE GOING TO GET WHERE YOU WANT TO GET AND BE ABLE TO IMPLEMENT WHERE YOU WANT TO GET IF YOU ARE TIED TOGETHER TO THESE FUNDING FORMULAS THAT HAVE A HISTORY YOU'RE SEEING IT EVERYWHERE YOU GO. SOME ARE GOING TO BIC INVESTIGATIVE, I'M SURE KNOWING SEATTLE THEY'RE INNOVATIVE, BUT SOME WANT TO HAVE A POT OF MONEY TO USE THE WAY THEY USED IN THE PAST AND AT THEIR DISCRETION. SO I HOPE YOU WILL P COP BACK TO -- COME BACK TO US OR WE GO TO YOU, WE NEED A POT OF MONEY WE ENCOURAGE THIS AND WE BACK YOU 100 P% THAT X PERCENTAGE OF THE CTSA BUDGET WILL BE USED AT DISCRETIONARY BASIS FOR BRINGING IN AND ALLOWING INNOVATION AND CREATION OF THE INFRASTRUCTURE, BECAUSE UNLESS YOU HAVE THAT YOU'RE NOT GOING TO GET WHERE YOU WANT TO GO. >> YOU TURN AROUND AND ASK YOU, YOU ARE THE ADVISORY COUNCIL WHAT WOULD YOU ADVISE WE DO? I TAKE YOUR POINT. IF YOU GO BACK TO THE IOM REPORT THEY TALKED ABOUT IT IN THE INNOVATION FUND. WHICH I LIKE AND DON'T LIKE, IN THE SENSE THAT IMPLIES PERHAPS WRONGLY BUT APPLIES I THINK EVERYTHING ELSE CTSA DO IS NOT INNOVATION. I WANT TO WHOLE THING TO BE ABOUT INNOVATION. BUT I THINK IT'S -- SO IS THAT SOMETHING YOU WOULD ADVISE US TO DO? (OFF MIC) >> I WOULD DEFINITELY AGREE WITH THIS. THIS IS SOMETHING WE STRUGGLED SINCE WE STARTED TO WORK WITH NCATS. WE'RE BASICALLY STUCK WITH PROCESSES AND DESIGN FOR SOMETHING ELSE, FOR ANOTHER ERA, FOR A TIME WHEN SCIENCE WAS DIFFERENT.8 AND WE HAVE NOT REALLY BEEN ABLE TO CHANGE THOSE THINGS. WE HAVE HEARD THE WAY THE SYSTEM IS MANIPULATED AND WE HAVE BEEN FRUSTRATED BY THE FACT THAT WE HAVE NOT BEEN ABLE TO BE AS TRANSFORMATIONAL AS OUR MISSION SEEMS TO IMPLY. UNTIL WE MANAGE TO CHANGE THE MECHANISM, SCIENCE CHANGES BUT FUNDING MECHANISM CONCERN FOR SCIENCE, WE SHOULD BE DOING EXPLORATION, ALL ABOUT TRYING NEW THINGS PUSHING THE ENVELOPE BUT MECHANISM DON'T ALLOW US TO DO THAT, SO WHAT CAN WE DO AS ADVISORY COUNCIL TO GET OVER THAT? AND CHANGE WHATEVER NEEDS TO BE CHANGE SO INSTEAD OF BEING FRUSTRATED BY -- FROM NOT BEING ABLE TO BE TRANSFORMATIONAL AS WE NEED TO BE, WE CAN FINALLY START MAKING PROGRESS ALONG THE LINE DESCRIBED WITH. >> I PERSONALLY WOULD LIKE TO SEE AN EVOLUTION OF A REVOLUTION. YOU CAN DEBATE WHETHER IT'S RHETORIC BUT THERE ARE MECHANISMS THAT DON'T WORK THE WAY WE DISTRIBUTE FUNDING. THIS HAS TO DO THE WAY WE MANAGE THE PROGRAMS. THERE ARE BETTER MANAGEMENT WAYS AVAILABLE, WE NEED TO IMPLEMENT THEM. ON THE OTHER HAND, IT DOESN'T MAKE SENSE TO MICROMANAGE TOO MUCH BECAUSE CENTERS AND PROGRAMS AND PROGRAMS HAVE EXPERTISE BRING TO THE TABLE AND WE WANT TO BUILD ON TO. SO I THINK DISCUSSION ABOUT REDEFINING PRIORITIES MAY LEAD TO REDISTRIBUTION OF FUNDS. I THINK THAT PROCESS CERTAINLY HAS TO BE TRANSPARENT. I DON'T THINK THAT CENTERS SHOULD HAVE TOTAL DISCRETION ABOUT HOW TO USE THE FUNDING AND AND NO ONE KNOWS HOW IT GOES. WE HAVE TO DO THAT IN PARTNERSHIP WITH THE STRATEGIC ALLIANCE, AND PARTICULARLY FROM MY POINT OF VIEW I'M A PUBLIC SERVANT P AND I SEE SIMILAR AS YOU SEE IT. SO BUT EXPERTISE IN TRANSPARENT FASHION IS VERY IMPORTANT. SO I WANT TO ECHO BOTH OF THOSE COMMENTS. BERNARD AND BOB IN PARTICULAR, INN INVESTIGATION FUNDS CERTAINLY A GREAT IDEA WE ARE TACKING REALLY BIG PROBLEMS AND OUR BUDGET WILL ALWAYS BE ORDER AFTER MAGNITUDE LOW THEY WERE SIZE OF THE PROBLEMS WE HAVE. GIVEN THAT'S THE CASE AND REALITY DOESN'T MEAN BIG PROBLEMS BUT I WANT TO REITERATE WHAT THE PREVIOUS COMMENTER MENTIONED IS WE NEED TO FIND ALLYIES AND THERE ARE LIKE MINDED GROUPS ALL AROUND OUR ECOSYSTEM WHO SHARE THE SAME INCENTIVES AND FOCUS. AND IF WE CAN LEVERAGE THEM THINK ABOUT SOME OF THE THINGS THAT WE'RE CONCLUDING WE NEED TO DO. A LOT HAVE TO DO WITH LARGE SCALE CULTURAL CHANGE. AND CHANGING INCENTIVE AND FUNDING HELPS BUT GOES QUICKER IF YOU HAVE MULTIPLE PEOPLE PUSHENING THE SAME DIRECTION. WHAT I ENCOURAGE TOWS DO IS IDENTIFY THOSE GROUP BUSINESS NAME AND FIND VERY DIRECT WAYS TO LEVERAGE THEIR EFFORTS AND OURS TOGETHER. TO MAKE IT THAT MUCH FASTER. >> WHO WOULD THOSE GROUPS BE? WHO COMES TO MIND IN YOUR MIND? >> WE WILL GO INTO IT FROM THE PHARMA BIOTECH PERSPECTIVE. CERTAINLY COLLEAGUES AND OTHER INSTITUTES EACH INSTITUTE SEEMS TO HAVE INNOVATIVE PROGRAMS IN THEIR OWN RIGHT THAT SEEM TO STAND ALONE, FUNCTION WELL BUT IF WE WORK TOGETHER WITH THEM NEURONEXT IS A GREAT EXAMPLE. BECAUSE YOU CAN DRIVE FORWARD, PHARMA BIOTECH IS ANOTHER ONE. CERTAINLY BIOTECH HAS A STRONG INCENTIVE TO SEE THIS VERY -- THIS VERY HIGH POTENTIAL INFRASTRUCTURE USE IN A WAY THAT'S TRANSLATIONAL. SO THERE'S LOTS OF DIFFERENT CONSTITUENCYINGS, I THINK YOU WILL FIND A LOT OF WILLING PARTIES TO HELP. >> YOU MAY HAVE NOTICED LYNN MARKS IS ON THIS WORKING GROUP. HE'S THE HEAD CLINICAL RESEARCH AT GSK BUT ALSO SPENDS LOTS OF TIME AS -- I FORGOT HIS OFFICIAL POSITION BUT OFFICIAL POSITION WITH N TRAN ACCELERATE AS WELL. WE HEARD THIS OVER AND OVER FROM HIM IN THE WORKING GROUP AND- CONVERSATIONS WITH HIM. SEPARATELY WHEN I VISITED GSK THAT A LOT OF THINGS WHICH THIS WORKING GROUP SEEMS TO BE SUGGESTING ARE THINGS THAT ARE QUITE CONGRUENT WITH WHAT -- IS TRYING TO DO SO THOSE ARE THINGS WE WANT TO DO. WE CERTAINLY WILL TAKE THIS AS AN ACTION ITEM ON OUR PART TO GET BACK TO YOU ABOUT HOW -- WHAT WE NEED FROM YOU TO GET ANSWER DIRECTLY BOB'S REALLY IMPORTANT QUESTION THIS IS, I AM FEELING ENORMOUS URGENCY TO SEIZE THE MOMENT. THIS MOMENT DOESN'T COME VERY OFTEN. IF WE DON'T DO THIS RIGHT NOW IT WILL BE ANOTHER DECADE UNTIL WE TRANSFORM THIS SYSTEM STORE THE BENEFIT OF SCIENCE AND PATIENTS. SO I'M VERY COMMITTED TO THIS, I THINK WE WILL TAKE AS AN ACTION ITEM FOR -- TO GET BACK TO YOU WHAT WE NEED AND NEEDED A VICE FROM YOU AS WE GO FORWARD. Q. SEEMS TO ME TRANSFORMATION YOU'RE LOOKING FOR WOULD BE ACCOMPLISHED THROUGH THE EVALUATION PROCESS. SO YOU SAID PETRA, IT'S THE RESPONSIBILITY BUT I BELIEVE IF YOU SET OBJECTIVES AND THEY'RE CLEAR AND IN RESPONSE TO THE IOM REPORT, THEN YOU SET UP AN EVALUATION PROCESS TO ENSURE THE PROGRAMS ARE MEETING. THE EXPECTATIONS. THAT TRANSFORMATION IS RAPID. >> >> CAN I ACD THAT I VERY MUCH APPRECIATE THIS THOUGHTFUL FEEDBACK AND PERSPECTIVE ON THE IOM REPORT. I THINK IT'S JUST AN -- EXCELLENT FOR ME TO START AT THIS POINT HAVING ALL THIS PERSPECTIVE. IT IS CORRECT THAT OF COURSE ANY CHANGE HAS TO BE DONE WHEN WE WOULD LIKE TO IMPLEMENT EVERYTHING AT ONCE HAS TO BE DONE, CAUTIOUSLY WITHIN THE ECOSYSTEM AND ENVIRONMENT AND I APPRECIATE YOUR HELP AND I HOPE YOU WILL BE AVAILABLE FOR FUTURE DISCUSSIONS AN FEEDBACK. >> I WILL JUST SAY COMPLETELY INDEPENDENTLY OF THIS WONDERFUL REPORT, IT WAS A VERY INTERESTING EDITORIAL -- CTSA PI AT INDIANA AND A -- ACTUALLY CURRENT HEAD OF THE ASSOCIATION FOR CLINICAL TRANSLATIONAL SCIENCE, JOINING OUR COUNCIL ACTUALLY IN SEPTEMBER. SO YOU WILL MEET HIM IF YOU HAVEN'T ALREADY. ON HIS OWN HE WROTE THIS QUITE REMARKABLE PIECE IN TRANSLATIONAL MEDICINE FROM PERSPECTIVE OF CTSA PI ADVOCATING MANY THINGS AND POINTING OUT MANY OF THE PROBLEMS, ADVOCATING MANY THINGS THAT THIS -- THE IOM REPORT STEERING COMMITTEE SAID. AND PUTTING OUT A LOT OF PROBLEMS THAT BOB TALKEDDED ABOUT. INTERESTINGLY, SO MY SENSE IS TRANSFORMATIONAL CHANGE HAPPENS WHEN ENOUGH PEOPLE GET SO FRUSTRATED BY THE CURRENT SYSTEM THAT IT OVERWHELMS THE UNDERSTANDABLE DESIRE TO KEEP DOING WHAT YOU'RE ALREADY DOING. MY SENSE IS WE'RE BEYOND TIPPING POINT, WE DON'T NEED TO SEIZE THE DAY, NOT EASY WE'RE CERTAINLY GOING TO LOOK TO YOU FOR YOUR HELP AND COUNCIL AS WE MOVE FORWARD TO THIS NEXT STAGE AND IMPLEMENTING THIS REPORT. IT'S WHAT WE HAVE BEEN WAITING FOR BEFORE WE WROTE THE NEW SET OF RFAs BECAUSE WE DIDN'T WANT TO DO THAT WITHOUT KNOWING WHAT THE RECOMMENDATIONS WERE. AND SO PETRA IS NOT GOING TO BE -- SHE'S NOT GOING HOME THE NEXT THREE MONTHS. NOW THAT SHE HAS THE REPORTS. I DON'T KNOW IF YOU TOLD YOUR H HUSBAND YET BUT YOU MIGHT WANT TO -- HE'S RIGHT THERE. OKAY. ALL RIGHT. SO THANK YOU AGAIN TO YOU THREE. WE HAVE A BREAK UNTIL 1:30 FOR LUNCH. THERE IS THE USUAL SET OF CULINARY DELIGHTS DOWNSTAIRS. THE THREE CO-CHAIRS ARE GOING TO BE DOING A MEDIA AVAILABILITY NOW BUT SO THEY MAY NOT JOIN YOU BUT WE WILL SEE YOU BACK HERE AT 1:30. CAN WE START EARLIER? OKAY. WE CAN START EARLIER I'M TOLD. SO WHAT DO YOU THINK, 1:15? ALL RIGHT? IN 1:15. COOL. ALL RIGHT. THANK YOU. >> SO WE'RE GOING TO GET STARTED AGAIN, AND FREDA AND JEFF ARE GOING TO TALK TO US ABOUT THE CURES ACCELERATION NETWORK. I DON'T KNOW WHETHER -- I THINK DAN IS -- WHERE IS DAN? >> RIGHT THERE, READY TO GO FIRST. DAN ALWAYS IS. >> DAN IS GOING TO TELL US -- WHY DON'T YOU SAY WHAT'S GOING ON. THIS IS YOUR PART OF THE PROGRAM. >> OKAY, I CAN DO THAT. SO THIS PART OF THE MEETING IS REALLY A LOOK AT SOME OF THE WORK THAT'S HAPPENED WITH CAN RECENTLY. SO THE FIRST THING, AND ACTUALLY YOU MENTIONED IT EARLIER, THAT DAN IS GOING TO WALK US THROUGH THE PROCESS FOR TRANSITION TO THE SECOND PHASE OF ORGANS-ON-CHIPS PROGRAM, SO THIS IS TO REALLY TALK WITH WHAT THE TRANSITION WILL LOOK LIKE. THEN WE'RE GOING TO TALK ABOUT PRIORITIZATION OF CAN PROJECTS AND FUNDING OVER THE NEXT YEAR OR SO, LOOKING FORWARD TO THE 2015 FUNDING YEAR. SO I'M JUST GOING TO KICK US OFF BY REMINDING US WHAT THE MISSION AND VISION FOR CAN ARE, AND WHAT OUR REMIT IS, AND THEN JEFF GINSBURG IS GOING TO WALK US THROUGH SOME IDEAS AND LEAD US IN A VERY ACTIVE DISCUSSION, WE HOPE, ABOUT WHAT SOME OF OUR AREAS OF PRIORITY SHOULD LOOK LIKE NEXT YEAR. >> [INAUDIBLE] [LAUGHTER] SO FOR THOSE OF YOU THAT ARE LISTENING IN, WE'RE JUST GETTING THE SLIDES UP AND MOVING, AND WE WILL BE READY TO ROCK IN JUST A MINUTE. >> I THINK WE'RE READY. SORRY FOR THE DELAY. AS FREDA SAID, THIS IS PRESENTING THE PLAN FOR NCATS OH TO TRANSITION FROM THE FIRST PHASE OF THE PROGRAM ON ORGANS-ON-CHIPS AT THE SECOND PHASE, AND I THINK CHRIS ALREADY GAVE AN INTRODUCTION IN TERMS OF SOME OF THE UPDATES TO COUNCIL DURING THE DIRECTORS UPDATE REPORT, BUT JUST TO GIVE YOU A BRIEF OVERVIEW AGAIN OF THE ORGANS-ON-CHIPS PROGRAM, MICRO PHYSIOLOGICAL SYSTEMS PROGRAM, ESSENTIALLY OUR GOAL OVER THE PIEF YEARS IS TO DEVELOP AN IN VITRO PLATFORM THAT USES HUMAN TISSUES TO EVALUATE THE EFFICACY, SAFETY AND TOXICITY OF THERAPEUTICS. THE GOAL IS TO CREATE 10 HUMAN SYSTEMS, THE CIRCULATORY, ENDOCRINE, GASTROINTESTINAL, IMMUNE, MUSCULOSKELETAL, NERVOUS, REPRODUCTIVE, RESPIRATORY, URINARY,, WE WANT IT TO BE RELEVANT, DIVERSE AND ALSO MODEL HUMAN DISEASES, AND ESSENTIALLY THE PLATFORM NEEDS TO BE MODULAR AND THE CELLS SURVIVING FOR AT LEAST FOUR WEEKS. THIS IS A JOINT PROJECT BETWEEN NIH, DARPA AND THE FDA, WITH NIH AND DARPA CONTRIBUTING ABOUT $75 MILLION EACH OVER THE COURSE OF THE FIVE YEARS OF THE PROGRAM. NIH IS ALSO GETTING SUPPORT -- THE PROGRAM IS ALSO GETTING SUPPORT FROM OTHER ICs LIKE NIDIB, NIEHS, CHILD AND HUMAN DEVELOPMENT AND -- SORT OF LIKE A MULTI-INSTITUTE SUPPORT ACROSS THE NIH IN ADDITION TO SUPPORT FROM THE COMMON FUND. SO MAJORITY OF THE SUPPORT THAT IS CONTRIBUTED BY NCATS IS COMING FROM THE CURES ACCELERATION NETWORK ALLOCATION OF MONEY. AND IN THIS PARTICULAR CASE, THE FDA PROVIDES REGULATORY GUIDANCE TO OUR PIs SO THAT THEY CAN DEVELOP THE PLATFORMS WITH THE VIEW IN MIND THAT THIS POTENTIALLY CAN BE USED AS REGULATORY TOOLS FOR DECISION-MAKING IN REGULATORY SCIENCE. AND SO IN THE TIMELINE I'M SWING HERE, WE ARE AT THIS TIME POINT IN YEAR TWO, GOING INTO YEAR TWO, AND DURING THE FIRST TWO YEARS, THE GOAL OF THE PROGRAM WAS TO DEVELOP CELL SOURCES, AND THIS WAS DONE THROUGH THE U18, ESSENTIALLY TO DEVELOP RENEWABLE CELL SOURCES PRIMARILY FROM INDUCED -- STEM CELLS AS WELL AS PROGENITOR CELLS, AND ALSO TO COME UP WITH THE PLATFORMS, BIOENGINEERING PLATFORMS WHICH CAN SUPPORT VIABILITY OF THE CELLS OVER THE COURSE OF FOUR TO SIX WEEKS OR MORE. AND ALSO TO BE ABLE TO DO SOME OF THE READOUTS HA WE NEED TO ASSESS SAFETY AND EFFICACY. NOW WHAT WE ARE GOING MOO AND WHAT WE'RE LOOKING POORD MOO THE SECOND PHASE WOULD REALLY BE TO INTEGRATE THE PLATFO SO WHAT WE HAVE RIGHT NOW ARE INDIVIDUAL ORGAN CHIPS. AS CHRIS SHOWED IN THE SHOW AND TELL, WE HAVE AN INDIVIDUAL BLOOD BRAIN BARRIER IN THE CHIP, SO EVENTUALLY WHAT WE WOULD LIKE IS TO LINK UP ALL THOSE MODULES SO THAT WE WOULD HAVE THE HUMAN BODY AND THE CHIP ALL LINKED TOGETHER AND IN A CONFIGURE RABL FORM THAABLEFORM THAT YOU CAN LINE UP THE ORGANS ON CHIPS DEPENDING ON THE BIOAVAILABILITY OF THE DRUG THAT ONE IS TESTING. SO THIS IS SORT OF LIKE A ONE SLIDE SUMMARY OF WHAT HAS BEEN ACHIEVED IN THE PROGRAM. I WON'T GO INTO A LOT OF THE DETAILS BUT JUST TO HIGHLIGHT THE PROGRAM, SO THE PROGRAM IS REALLY -- THE INVESTIGATORS WITHIN THE PROGRAM HAVE REALLY REACHED MOST OF THEIR MILESTONES. THERE ARE STILL A FEW THAT MEET THEIR MILESTONES IN THE NEXT TWO MONTHS, THE OFFICIAL END DATE OF THE PROJECT IS AT THE END OF JUNE, SO THEY HAVE ESSENTIALLY ABOUT A MONTH AND A HALF OR SO TO FINISH ALL THEIR MILESTONES, BUT I CAN SAY MAJORITY OF THE MILESTONES HAVE BEEN MET. THEY HAVE DEMONSTRATED FUNCTIONAL AND PHYSIOLOGICAL RELEVANCE OF THE ORGANS-ON-CHIPS, THEY HAVE BEEN TESTED WITH A SET OF COMPOUNDS WITH KNOWN TOXICITIES AND HAVE BEEN SHOWN TO HAVE THE PREDICTED TOX TIS IT RESPONSE FROM EACH OF THE INDIVIDUAL ORGAN SYSTEMS. VIABILITY HAS BEEN SHOWN TO RANGE ANYWHERE FROM 3 TO 6 WEEKS, IN VIVO-LIKE ABSORPTION, EXCRETION AND ENZYMATIC ACTIVITY, AS WELL AS NONINVASIVE AND NON-DESTRUCTIVE READOUTS OF THIS. AND THE INVESTIGATORS ARE ALSO STARTING TO USE INDUCED STEM CELLS AS WELL AS PROGENITOR CELLS. GOING TOWARDS THE TOOLS HAVE BEEN MADE AVAILABLE TO INVESTIGATORS WITHIN THE CONSORTIUM. WE HAVE DEVELOPED QUALITY CONTROL AND CHARACTERIZATION OF THE STEM CELLS VIA THE CORIELL REPOSITORY AND THE STEM CELLS A BEING MADE AVAILABLE TO INVESTIGATORS. WE HAVE MADE AVAILABLE A TRAINING SET OF COMPOUNDS TO TEST THE ORGAN SYSTEMS THROUGH A CONTRACT WITH EVOTEC. THE INVESTIGATORS HAVE DEVELOPED, AS YOU SAW IN THE DEVICE THAT WAS SHOWN EARLIER, MINI PUMPS, MICRO ANALYZERS, TOXICITY DATABASE, AS WELL NON-DESTRUCTIVE BIOSENSORS TO ALLOW US TO HAVE QUICK AND EASY READOUTS IN TERMS OF THE PHYSIOLOGICAL RESPONSE, AS WELL AS ALSO BICOMPATIBLE SCAFFOLDS TO SUPPORT THE 3-D ORGAN SYSTEM DEVELOPMENT. NOW WHAT WE ARE INFORMING THE C.A.N. REVIEW BOARD AS WELL AS COUNCIL IN TERMS OF WHAT HAS BEEN DONE TO DETERMINE WHICH OF THE PROJECTS WILL MOVE FORWARD, THERE WILL BE SOME DOWN SELECTION OF THE PROJECT AND SOME STREEN STREE STREAM THE PROJECT TO MAKE SURE WE'RE JUST MOVING THE BEST SCIENCE FORWARD, SO HERE WE ARE GOING INTO THE END OF JUNE -- OR A TRANSI DATE, SO IN THAT PROCESS, THE ORDs HAVE SUBMITTED A PROGRESS REPORT AS WELL AS ANY PROPOSED CHANGES TO THE US3, WHICH IS THE NEXT THREE YEARS OF THE AWARD. THE EVALUATIONS HAVE BEEN ADMINISTRATIVELY BY REVIEWERS THAT HAVE BEEN PULLED OUT FROM THE TRANSNIH MICRO PHYSIOLOGICAL SYSTEMS PROJECT TEAM, SO THEY ARE ESSENTIALLY THE EXPERTS IN ORGAN FUNCTION AND DEVELOPMENT FROM EACH OF THEIR RESPECTIVE INSTITUTES AT THE NIH. WE HAD TWO TO THREE REVIEWERS FOR EACH OF THE AWARDS THAT WERE MADE. THE SCORING METRICS THAT WE USED WAS ESSENTIALLY DERIVED FROM THE CRITERIA THAT THE C.A.N. REVIEW BOARD CAME UP WITH, IF YOU REMEMBER BACK IN -- ABOUT A YEAR AGO, MAY 2013, IN TERMS OF WHAT ARE THE METRICS THAT THE PROGRAM SHOULD BE EVALUATED BY. AND THEN WE USE A RANKING IN TERMS OF THE PRIORITIES BY WHICH THE AWARDS ARE BEING EVALUATED, SO WE PUT MILESTONE PROGRESS A TOP PRIORITY, THAT THE PIs -- THE INVESTIGATORS WHO MET THEIR MILESTONES ARE GOING TO BE GIVEN A LOT OF POINTS FOR THAT. WHAT THEY HAVE DONE WITH THE SUPPLEMENT MONEY THAT WE HAD GIVEN THEM, THE VALIDATION OF INDIVIDUAL ORGAN SYSTEMS, ESSENTIALLY THE REPRODUCIBILITY OF THE PLATFORMS, THE KIND OF COMPOUNDS THAT THEY TESTED, THE MECHANISM OF ACTION THAT CLASSES THE COMPOUNDS, FUNCTIONAL ASSAYS, WE ALSO LOOKED IN TERMS OF THE COMPLEXITY OF THE ORGAN SYSTEMS AND ESSENTIALLY COLLABORATIONS BOTH WITHIN AND OUTSIDE THE CONSORTIUM, WE PLACED PARAMOUNT IMPORTANCE IN TERMS OF FOSTERING EXCHANGE OF RESOURCES AND MATERIALS AND REALLY MAKING SURE THAT THE PROGRAM PRODUCES THE BEST ORGAN SYSTEMS -- ORGANS-ON-CHIPS WITHOUT A LOT OF REDUNDANCIES. THEN THE NEXT ONE IS THE EVALUATION OF THE U18 AWARD. THESE ARE ESSENTIALLY TWO-YEAR AWARDS SO TECHNICALLY THEY WOULD END AT THE END OF JUNE. BUT THE INVESTIGATORS DEVELOPED VALUABLE RESOURCES TO THE PROGRAM AND I THINK CERTAINLY TO THE GENERAL SCIENTIFIC COMMUNITY IN TERMS OF THE STEM CELLS, THE KIND OF RENEWABLE CELL SOURCES THAT ARE NECESSARY TO SEED INTO OUR PLATFORMS. WE WILL ALSO ASKING -- WE HAVE ASKED THE COMMON FUND TO PROVIDE US WITH ADDITIONAL MONEY, AND WE ARE ABLE TO PROVIDE SUPPLEMENT TO THESE INVESTIGATORS TO CARRY THEM INTO WORK THAT WOULD ALLOW THEIR CELL SOURCES TO BE INTEGRATED WITH THE THREE YEARS OF -- INTO THE THREE YEARS OF THE PROGRAM, SO THE INVESTIGATORS ARE MOVING FORWARD INTO THE THREE YEARS, ARE COLLABORATING WITH THE U18 INVESTIGATORS, INCORPORATING THE CELL SOURCES, AND WE ARE THEN PROVIDING THEM WITH THE FUNDS TO MAKE THAT TRANSITION SO THAT THEIR CELLS ARE ESSENTIALLY UTILIZED IN THE MAXIMUM WAY. SO WE THEN FACTORED IN ALSO IN TERMS OF THE REVIEW THEIR MILESTONES, THE PROGRESS AND VALIDATION, REPRODUCIBILITY, AND THEN ASKED THEM AT THEIR COMPLETION, THEIR CELLS SHOULD BE DONE WITHIN ONE YEAR OF SUPPLEMENT MONEY. SO WE CAN GO INTO DETAILS IN TERMS OF THE INDIVIDUAL APPLICATIONS OR SUPPLEMENT REQUESTS IN CLOSED SESSION, BUT THIS IS JUST TO GIVE YOU A GENERAL OVERVIEW OF THE PROCES THAT WE HAVE GONE THROUGH IN EVALUATING THE INDIVIDUAL REQUEST FOR SUPPLEMENTS AS WELL AS THE PROJECTS AS THEY GO INTO THE NEXT PHASE. >> DAN, THAT WAS VERY GOOD. COULD YOU JUST GIVE, FOR THE C CAN AND THE C.A.N. BOARD'S BENEFIT, REVIEW FOR THEM HOW A UHTU, UH3 SYM WORKS, THE FACT THAT THE REVIEWERS ARE -- INTERNAL REVIEWERS, NOT EXTERNAL REVIEWER, ALL THAT KIND OF STUFF? THEY MAY NOT BE FAMILIAR WITH THIS WHOLE IDEA. >> RIGHT. SO WHEN THIS PROGRAM STARTED TWO YEARS AGO, THE ENTIRE APPLICATION PACKAGE, WHICH MEANS THE PROPOSED WORK FOR BOTH THE UH2 PHASE AND THE UH3 PHASE HAVE BEEN ALL LAID OUT BY THE INVESTIGATORS AND THAT HAS GONE THROUGH PEER REVIEW BY CSR, SENT THERE FOR SCIENTIFIC REVIEW, ACTUALLY WENT THROUGH THE NIDDK ADVISORY KUN BECAUSE WE DIDN'T HAVE A FORM, NCATS ADVISORY COUNCIL AT THAT TIME. SO THEY HAD REVIEWED THE APPLICATIONS PROVIDING SECOND LEVEL REVIEW IN CONCURRENCE WITH THE EVALUATION THAT WAS DONE BY PEER REVIEW, SO THE UH2, UH3 PHASES ARE NOW JUST BEING LOOKED AT THE SECOND TIME ADMINISTRATIVELY BY -- INTERNALLY BY NIH STAFF. WHAT THAT ACHIEVES IS THAT WE JUST LOOK AND REVISIT WHAT WAS ALREADY PEER REVIEWED, ALLOWING THE INVESTIGATORS TO UPDATE THEIR GOALS BASED ON WHAT HAS CHANGED IN TECHNOLOGY, CELL SOURCES, AND ESSENTIALLY TAKE INTO CONSIDERATION THE KIND OF INTERACTIONS THAT EACH OF THE INVESTIGATORS HAVE NOW HAD THE BENEFIT OF FUNCTIONING AS A CONSORTIUM. SO WE ALLOW THEM FLEXIBILITY TO TWEAK THEIR UH3 GOALS, SO AT THE END, WE SHOULD COME UP WITH THE BEST PRODUCT AND NOT JUST KIND OF HAVE THEM STAY ON TRACK WHAT WAS PROPOSED TWO YEARS AGO WITHOUT ANY CHANGES, SO WE ALLOW THEM TO HAVE THOSE CHANGES, SO THAT'S WHY THERE'S THE NEED TO DO THAT ADMINISTRATIVE REVIEW. ALSO WE WANTED TO EVALUATE HOW THEY HAVE DONE AS A GROUP AND INDIVIDUALLY IN THE PAST TWO YEARS OR SO. ANY OTHER QUESTIONS OR COMMENTS? >> DAN, YOU'VE GOT TO TALK ABOUT THIS AGAIN IN THE CLOSED SESSION? >> BRIEFLY. AGAIN, IT WOULD BE -- UNLESS THERE ARE SPECIFIC QUESTIONS ABOUT INDIVIDUAL APPLICATIONS, IT WILL BE IN THE ELECTRONIC COUNCIL BOOK, EACH INDIVIDUAL APPLICATIONS ARE IN THE ELECTRONIC COUNCIL BOOK, SO UNLESS A COUNCILMEMBER WANTS TO TALK ABOUT THE APPLICATIONS INDIVIDUALLY, I CAN TALK INDIVIDUALLY ABOUT THEM. IF NOT, WE'LL GO THROUGH OUR REGULAR CONCURRENCE AND CLOSED SESSION. FRANK, YOU HAD A QUESTION? ANYTHING ELSE? IF NOT, THEN -- >> WHILE FREDA IS GETTING GOING, I JUST WANT TO COMMENT THAT YOU MAY BE WANTING TO HEAR MORE OF THE SCIENCE THAT ACTUALLY WENT ON HERE BECAUSE THERE IS A HUGE AMOUNT OF REALLY INTERESTING SCIENCE THAT WENT ON IN THE UH2 PROGRAM THAT ALLOWS US TO MOVE ON TO THE UH3. THAT WASN'T THE PURPOSE OF DAN'S PRESENTATION THIS TIME, IT'S TO GO OVER FOR YOU THE FACT THAT WE ARE MOVING FROM UH2, UH3, WE HAVEN'T HAD DAN TALK ABOUT THE PROGRAM IN GENERAL SCIENTIFICALLY IN A WHILE, SO THAT'S SOMETHING WE'LL DO IN AN UPCOMING COUNCIL MEETING. >> I'M GLAD YOU MENTIONED THAT. YOU MOA, DAN STOO KNOW, DAN STOOD UP HERE AND MADE IT SOUND SO EASY AND SO STRAIGHTFORWARD. THIS IS JUST A BRILLIANT PIECE OF WORK WITH INCREDIBLE ADVANCEMENT IN A VERY SHORT PERIOD OF TIME, AND THE INCREDIBLE SCIENTISTS THAT ARE INVOLVED IN IT ARE A PART OF IT. I THINK DAN AND CHRIS' LEADERSHIP ARE BOTH INCREDIBLE CONTRIBUTIONS TO MOVING THIS WORK FORWARD QUICKLY AS WELL. SO WHAT WE WANT TO DO NOW, YOU KNOW, THE TISSUE ON A CHIP PROGRAM IS BASICALLY OUR MAJOR EXPENDITURE OUT OF THE C.A.N. BUDGET. WE HAVE A COUPLE OF ACCOUNTABILITIES AS A C.A.N. REVIEW BOARD, AND WE WANTED TO MAKE SURE THAT WE REMINDED OURSELVES OF WHAT THAT WAS, AND THEN TO SPEND AS MUCH TIME AS WE CAN KEEP AVAILABLE TO OURSELVES TO DISCUSS HOW WE WOULD PRIORITIZE WORK TO MAKE THOSE RECOMMENDATIONS TO NCATS AND NCATS LEADERSHIP IN THE FORM OF CHRIS, SO I'M GOING TO TAKE THE OPPORTUNITY TO QUICKLY REMIND US OF OUR MISSION, VISION AND REMIT, AND OUR TRANSACTION AUTHORITY, AND THEN JEFF GINSBURG IS GOING OH TO WALK US THROUGH SOME AREAS OF FOCUS THAT I'M HOPING AS A GROUP WE CAN HAVE ROBUST DISCUSSION AROUND, AND THEN HONE IN ON SOME THAT WE THINK ARE WORTHY OF FURTHER DEVELOPMENT, AND THEN SHOOT FOR A RECOMMENDATION. SO AGAIN, I'M GOING TO REFRESH YOU ON THE C.A.N. MISSION. I'M GOING TO REMIND OURSELVES OF THE CURRENT REPORT LANGUAGE, WHICH IS ACTUALLY GOOD NEWS BIT OF LANGUAGE, THEN WE'LL PURSUE THE DISCUSSION. SO THE BACKGROUND AGAIN IS THAT THE C.A.N. R.B. IS MEANT TO ADVISE AND PROVIDE RECOMMENDATIONS TO THE DIRECTOR, AND THAT'S BOTH WITH RESPECT TO WHAT THE ACTUAL BARRIERS ARE, SO A REAL LANDSCAPE IN POINTING OUT, IF YOU WOULD, OF WHAT CURRENT BARRIERS, KNOWN BARRIERS ARE, AND TO ALSO PROVIDE RECOMMENDATIONS ON POLICIES, PROGRAMS AND PROCEDURES. AT THE MEETING IN JANUARY, WE REVIEWED THE THREE KIND OF PREVIOUS TRANSLATIONAL LANDSCAPING EXERCISES, TALKED A LITTLE BIT ABOUT THAT THEN, AND WE PROMISED THAT WE WOULD COME BACK AT THIS MEETING WITH A LITTLE BIT OF A SYNTHESIS OF THOSE AND SOME RECOMMENDATIONS OF TOPICS IT PUSH ON, AND THAT'S WHAT JEFF IS GOING TO LEAD US THROUGH. SO IF THAT'S OUR KIND OF REMIT, WHAT ARE OUR FUNCTIONS? SO AS A C.A.N. FUNCTION, WE CONDUCT AND SUPPORT REVOLUTIONARY ADVANCES IN BASIC RESEARCH, TRANSLATING, YOU KNOW, I GUESS TACKLING THE BASIC SCIENTIFIC ISSUES IN TRANSLATIONAL RESEARCH. HOW DO YOU GET FROM A SCIENTIFIC DISCOVERY TO CLINICALLY MEANINGFUL OUTPUT? THERE'S AN AWARD OF GRANTS AND CONTRACTS, SO THAT'S WHY WE'RE GOING TO TAKE A CHANCE TO TALK A LITTLE BIT ABOUT THE AUTHORITY TO SEE IF WE CAN MORE CLEVERLY LEVERAGE THE AUTHORITY THAT IS AVAILABLE TO US, AND TO ALSO PROVIDE RESOURCES TO CONVENE, IF YOU WOULD, AND PROVIDE AN INTERFACE AND AN INTEGRATION ACROSS THE ECOSYSTEM TO ADVANCE HIGH NEED CURES, AND THEN LAST BUT NOT LEAST, AFTER WE'VE IDENTIFIED THOSE BARRIERS AND MADE RECOMMENDATIONS TO DO WORK THAT REDUCES HOS THOSE BARRIERS AND TO FACILITATE THE REGULATORY PATHWAYS IN THAT SPACE, SO IT'S REALLY THE ENTIRE SPECTRUM OF ACCELERATING TRANSLATIONAL WORK. AND THEN VERY QUICKLY TO GO BACK OVER THE FUNDING MECHANISMS, RIGHT NOW WE HAVE THE AUTHORITY FOR AWARDS UP TO $15 MILLION PER PROJECT FOR THE FIRST PHYSICAL YEAR, AND WE HAVE THE POTENTIAL FOR ADDITIONAL FUNDING IN SUBSEQUENT YEARS. THERE'S AN OPTION FOR MATCHING FUNDS, SO THIS WOULD BE A PROVISION THAT WOULD ALLOW THAT AN ELIGIBLE ENTITY COULD ACTUALLY CONTRIBUTE IN A 1 TO 3 RATIO FOR A PROJECT THAT WOULD MOVE FORWARD. SO PUT A PIN IN THAT ONE, BECAUSE I THINK WE WANT TO THINK ABOUT SOME FUTURE PROJECTS AGAIN, OUR FUNDING. THEN LAST BUT NOT LEAST IS THE FLEXIBLE RESEARCH AUTHORITY. AND THIS IS TO USE THE OTHER TRANSACTIONS AUTHORITY TO FUND PROJECTS IN ACCORDANCE WITH THE TERMS AND CONDITIONS OF THIS SECTION, AND THAT THESE AWARDS, AGAIN, ALLOW US SOME FLEXIBILITY BEYOND WHAT IS TRADITIONALLY USED. SO THIS AUTHORITY IS NOT TO EXCEED 20% OF OUR BUDGET, WHICH RIGHT NOW REPRESENTS A SMALL AMOUNT. IT'S ABOUT $2 MILLION IF YOU LOOK AT OUR 2013 BUDGET, BUT AS WE MOVE FORWARD, WE HOPE THAT THAT NUMBER CONTINUES TO GROW. SO JUST QUICKLY, THE REPORT LANGUAGE AS IT CURRENTLY SAYS, FOR FY15, THE PRESIDENT PROPOSED THE FOLLOWING: $29.8 MILLION. YOU SHOULD KNOW THAT THIS REPRESENTS A SIGNIFICANT INCREASE OVER 2013 AND 2014 DOLLARS. 20 MIL TO BE EXACT, OVER '14. AND THE INCREASE IS PARTIALLY TO FUND THE WONDERFUL PROGRAM THAT DAN JUST REMINDED US OF, WHICH IS MOVING NICELY ALONG. BUT THERE ARE OTHER FUNDS THAT WOULD BE AVAILABLE AND WE'RE HOPING THAT THAT WILL BE THE FOCUS OF THE DISCUSSION THAT WE HAVE HERE IN JUST A MINUTE. AND REMEMBER THE PROJECT CRITERIA IS THAT IT'S COLLABORATIVE, IT HAD DISCRETE AND MEASURABLE OUTCOMES, THAT WE HAVE BROUGHT IN SIGNIFICANT IMPACT. IN FACT, I'D LIKE TO TALK ABOUT THIS AS KIND OF THE INTERSECTION BETWEEN THE BROADEST POSSIBLE IMPACT AND MEETING THE DEEPEST POSSIBLE NEEDS. AND THEN THE TIMELINE FOR COMPLETION IS -- SHOULD BE NO SHORTER THAN FIVE YEARS. SO I WON'T START TALKING ABOUT THIS OTHER PART OF IT. I'M GOING TO LET JEFF JUMP RIGHT INTO THE IDEAS FOR THE C.A.N. PROJECTS, BECAUSE HE'LL LET YOU KNOW HOW WE STARTED TO THINK ABOUT THIS IN GENERAL, AND THEN MORE SPECIFICALLY. SO WITHOUT FURTHER ADO, JEFF? >> THANKS, FREDA. ALSO I WANT TO THANK CHRIS FOR GIVING US CHRISTINE. SO SOME OF YOU MAY NOT KNOW THAT CHRISTINE CATILLO IS NOW ALSO WORKING WITH THE C.A.N. BOARD, AND IF SHE'S HALF AS EFFECTIVE AS SHE WAS WITH THE CTSA PROJECT THAT WE JUST HEARD ABOUT, WE'LL BE IN GREAT SHAPE. SO IT'S GREAT TO HAVE YOU ON BOARD. I ALSO WANT TO JUST REMIND US THAT WHAT WE'RE TALKING ABOUT HERE IS AN OPPORTUNITY THAT WE HAVEN'T HAD BEFORE TO PUT SOME REAL DOLLARS TOWARD SOME PROJECTS, SO OUR GOAL HERE IS TO TEE UP SOME IDEAS AND BY SEPTEMBER, SHOULD THE APPROPRIATION GO THROUGH, WE HOPE IT WILL, AS IT SEEMS LIKE LIE IT WILL, THAT WE'LL HAVE THINGS THAT WE CAN PULL THE TRIGGER ON FOR FUNDING. SO THE PROCESS BY WHICH WE GOT TO THIS POINT WAS MAINLY TO TAKE ADVANTAGE OF SOME OF THE PRIOR WORK THAT HAD BEEN DONE AT NIH AND NCATS THROUGH THE FOUR INITIATIVES, IF YOU WILL, OR REPORTS. THE NCATS STAFF HAS CULLED THROUGH SOME OF THOSE FOR US AND SELECTED ONES THAT LOOK LIKE THEY MIGHT BE ALIGNED WITH THE C.A.N. MISSION AND GOALS. AND THEN OVER THE LAST MONTH OR SO, FREDA, MYSELF AND CHRISTINE HAVE TRIED TO REDUCE THOSE TO A MORE MONNAGABLAMORE MANAGEABLE NUMBER. IT'S NOT A PERFECT PROCESS BY ANY MEANS, BUT WE ALSO TOOK WHAT YOU ALL -- WHAT THE BOARD SAID LAST TIME WAS LET'S TAKE ADVANTAGE OF THINGS THAT HAVE ALREADY BEEN TEED UP IN THE PAST AND START FROM THAT RATHER THAN TO CREATE DE NOVO IDEAS. ALTHOUGH THAT CONCEPT IS STILL ON THE TABLE. AND I ALSO WOULD LIKE YOU TO THINK ABOUT AS WE GO THROUGH SOME OF THESE PROCESSES, WHICH ONES, AS FREDA SAID, WILL HAVE THE OPPORTUNITY FOR THE GREATEST IMPACT FOR THE DOLLARS SPENT AND THAT ADDRESS SOME OF THE CRITICAL GAPS THAT WE'VE BEEN TALKING ABOUT IN TRANSLATION MALTRANSLATIONALSCIENCE. SO WITH THAT, LET ME START WITH THERE ARE GOING TO BE NINE OF THESE AREAS THAT WE'VE IDENTIFIED. THEY'RE OBVIOUSLY AS DEPICTED ON THESE SLIDES EXTREMELY HIGH LEVEL, AND THE GOAL WILL BE, ONCE WE HONE THESE DOWN TO A SMALLER NUMBER TO DRILL DOWN INTO GREATER DETAIL ON WHAT A PROJECT OR SERIES OF PROJECTS MIGHT LOOK LIKE THAT MIGHT BE OPPORTUNITIES FOR FUNDING. SO THIS FIRST ONE ENTITLED REGULATORY FOCUS AREA, I THINK ALIGNS ACTUALLY QUITE NICELY WITH SOME OF THE DISCUSSION THIS MORNING ON THE NEED FOR PERHAPS FDA TO BE MORE ENGAGED IN PERSONALIZED MEDICINE. CLEARLY, THEY ARE, AND HAVE BEEN VERY PROACTIVE, BUT I GUESS THE NOTION THAT THERE'S MORE TO BE DONE IN THIS SPACE IS SOMETHING THAT WE PROBABLY WOULD ALL AGREE ON IF WE'RE GOING TO STREAMLINE TRANSLATION. AND IN THIS PARTICULAR AREA, I THINK THE NOTION IS TO THINK ABOUT THE PATHWAY BY WHICH BIOMARKERS EITHER BECOME DIAGNOSTICS IN THEIR OWN RITE BECOME COMPANION DIAGNOSTICS THAT ENABLE THERAPEUTIC PROGRAMS TO HAVE POTENTIALLY A COMPANION DIAGNOSTIC ASSOCIATED WITH IT. SO I THINK THE IDEA FOCUSES AROUND WAYS IN WHICH NCATS AND FDA COULD PLAY A MORE SIGNIFICANT ROLE IN COORDINATING THE ROLE OF COMPANION DOOG DIAGNOSTICS PARTICULARLY WITH THERAPEUTIC PROGRAMS BEING UNDERTAKEN BY PHARMA AND OTHER BIOTECHNOLOGY COMPANIES THROUGH MECHANISMS SUCH AS SBIR, STTR, BUT ALSO THE NOAX THAT THERE ARE PROBABLY THERAPEUTIC PROGRAMS WITHIN NIH THAT WOULD BENEFIT FROM THE DEVELOPMENT OF BIOMARKERS THAT COULD EVENTUALLY EITHER INFORM THEIR TRANSLATIONAL ACTIVITIES OR POTENTIALLY BE PART OF A REGISTRATION TRIAL PORE APPROVAL, AND THEN A COMPANION DIAGNOSTICS. SO THIS IS ONE OF THE AREAS WHERE FDA, NCATS AND THE SCIENTIFIC COMMUNITY ENGAGED IN BIOMARKER RESEARCH COULD COME TOGETHER. I'LL GO THROUGH ALL OF THESE AND THEN I HOPE WE HAVE TIME FOR DISCUSSION. IT'S MANDATORY THAT WE DO. THE SECOND AREA HAS TO DO WITH PRECLINICAL ACTIVITIES AROUND COMPOUNDS THAT MIGHT HAVE ACTUALLY FAILED. IT SAYS HERE PARTIALLY DEVELOPED, BUT I THINK THE NOTION THAT WE WERE THINKING ABOUT WAS COMPOUNDS THAT HAVE ACTUALLY FAILED IN DEVELOPMENT, SO EXPANDING THE CURRENT REPURPOSING PROGRAM TO SOME SOME DEGREE AND USE THE ECK SITH CYSTING DATA, BUT INCREASING BEYOND THE NUMBER OF 58 IS SOMETHING WE THINK WOULD BE VERY ADVANTAGEOUS. SELECT WHICH OF THE BEST CANDIDATES FOR CLINICAL DEVELOPING FROM THESE, AND DO THIS IN A MUCH MORE COORDINATED WAY AND STREAMLINED WAY THAT WOULD PRO TENSIONALLY REDUCE THE BURDEN OF CURRENT CLINICAL TRIALS, IMPORTANTLY, TO LEVERAGE THE NTU PROGRAM IN THIS REGARD. I THINK OF THERE WAS ALSO A NOTION EMBEDDED IN HERE WHERE IT SAYS TOXICOLOGY AND PKs TO ALSO TAKE ADVANTAGE OF THE RAID PROGRAM ACTIVITIES AND ALSO BRING IN SOME OF THAT DATA THAT WOULD FACILITATE PRECLINICAL TO CLINICAL TRANSITIONS. SO THAT WAS THE SECOND IDEA. THE THIRD HAS TO DO WITH THE INEFFICIENCIES AND INADEQUACY BY WHICH WE KNOW ABOUT THE AVAILABILITY OF HUMAN BIOLOGICAL SPECIMENS. I'M SURE THIS HAS BEEN DISCUSSED HERE AS WELL AS THROUGHOUT NIH AS WELL AS EVEN IN THE CTSA PROGRAM, BUT IT SEEMS LIKE THERE ARE -- THAT THESE ARE ARE INCREDIBLY VALUABLE RESOURCES THAT COULD BE USED FOR BOTH TARGET DISCOVERY AS FOR BIOMEDICAL DISCOVERY, YET THERE ARE LACKS STANDARDS ACROSS THE BOARD FOR HOW SAMPLES ARE COLLECTED, MANAGED AND ACCESSED. MOST OF THE COLLECTIONS ARE SILOED WITHIN THE ORGANIZATIONS AND EVEN WITHIN THE INVESTIGATORS' LABORATORIES IN THE ORGANIZATIONS IN WHICH THEY RESIDE SO THEY SIT AS LATENT ASSETS THAT COULD POTENTIALLY BE MOBILIZED. SO THE NOTION OF POTENTIALLY HAVING M CAT NCATS TO CREATE THE NATIONAL BIOBANK, KNOWING WHERE SAMPLES ARE AND SOME METADATA AROUND THEM WOULD BE SOMETHING THAT WE COULD SEE AS POTENTIALLY ADVANTAGEOUS. AND THEN ALSO TO RELEASE THE VALUE OF THESE TO CREATE A MECHANISM PERHAPS AS INDICATED HERE, APPRISING MECHANISM, BUT EVEN A FUNDING MECHANISM, RFAs, TO DEVELOP NOVEL BIOMARKERS OR TO PURSUE PATHWAY-RELATED RESEARCH USING THESE VERY PRECIOUS SAMPLES THAT ARE GENERALLY LINKED TO IMPORTANT CLINICAL PHENOTYPES. THE FOURTH IDEA CENTERS AROUND THE ABILITY TO BRING FORWARD REAGENTS THAT COULD BE USED AGAIN FOR BIOMARKERS OR DIAGNOSTICS. THIS SLIDE INDICATES THAT THE FOCUS WOULD BE ON RARE AND NEGLECTED DISEASES, BUT ONE COULD EQUALLY ARGUE THAT THE SAME COULD BE SAID FOR MORE COMMON CHRONIC DISEASES, BUT NCATS COULD -- AND C.A.N. COULD GET BEHIND THE CREATION OF A PIPELINE FOR THE PRODUCTION OF QUALITY HIGH AFFINITY REAGENTS THAT COULD BE USED IN PRECLINICAL SETTINGS AS WELL AS ASSAY PLATFORMS TO CONVERT THESE AFFINITY REAGENTS AND TO BONA FIDE DIAGNOSTICS. WITH A GOAL OF ENABLING THE BETTER USE OF BIOMARKERS FOR MONITORING RESPONSE TO THERAPY PARTICULARLY IN THE RARE AND NEGLECTED DISEASES. THE FIFTH IDEA, AND PERHAPS THIS IS ALMOST -- COULD POTENTIALLY BE A NO-BRAINER, IS TO USE SOME OF OUR FUNDING MECHANISMS TO HELP INVESTIGATORS THAT HAVE HIT UP AGAINST A PARTICULAR TRANSLATIONAL BARRIER TO ALLOW THEM TO MOVE PAST THAT BARRIER AND ACHIEVE A STEP UP IN VALUE OF THE PROGRAMS THAT THEY'RE PURSUING. SO THIS COULD POTENTIALLY BE VIEWED AS A VALLEY OF DEATH TYPE OF PROGRAM WHERE THERE'S NOT THE OBVIOUS SOURCES AND RESOURCES TO DO, LET'S SAY A PRECLINICAL ASSAY DEVELOPMENT ACTIVITY OR TOX STUDY MAY NOT BE AVAILABLE, BUT PERHAPS A MECHANISM SUCH AS THIS COULD ALLOW SOME OF THE INVESTIGATORS HA AR THAT ARE REALLY FOCUSED ON THERAPEUTIC OR DIAGNOSTIC DEVELOPMENT TO OVERCOME WHAT MIGHT BE SEEMINGLY AN INSTRUMENTA MONUMENTAL BARRIER WITH A LIMITED AMOUNT OF RESOURCES. THE SIXTH AREA THAT WE TALKED ABOUT WAS -- CENTERED AROUND COLLABORATIONS AND PARTNERSHIPS, PARTICULARLY AROUND PHASE TWO STUDIES THAT PHARMACEUTICAL COMPANIES MIGHT BE PURSUING. I THINK THIS NOTION ALSO CAME, I THINK, FROM THE AREA OF CANCER-RELATED THERAPEUTIC DEVELOPMENT, WHERE THE POSSIBILITY OF DOING COMBINATION STUDIES IN A MUCH MORE COORDINATED WAY THAT ALLOWS FOR BOTH SAFETY AND EFFICACY EVALUATIONS. THIS IS SOMETHING THAT C.A.N. AND NCATS MIGHT BE ABLE TO GET BEHIND AND PERFORM THAT COORDINATION STUDY. AND SECONDLY, THE NOTION THAT WHEN MOLECULES ARE GOING INTO MAN FOR THE FIRST TIME, THERE'S A REAL OPPORTUNITY TO DO SYSTEMS PHARMACOLOGY, PARTICULARLY IF WE TAKE SOME UNBIASED APPROACHES USING GENOME-EN TECHNOLOGIES SUCH AS TRANSCRIPTIONAL ASSAYS OR WHOLE PROTEOME ASSAYS, ONE HAS AN OPPORTUNITY TO LOOK AT ALL THE POTENTIAL ME CAN MISM MECHANISMS AND PATHWAYS THAT A DRUG IS POTENTIALLY HITTING RATHER THAN THE ONES THAT WERE USED TO DEVELOP THE COMPOUND IN THE FIRST PLACE. SO I THINK THERE'S AN OPPORTUNITY HERE TO BEGIN TO THINK ABOUT EARLY SIGNALS OF EFFICACY THAT -- AND PARTICULAR MAYBE EVEN IN AREAS THAT WERE NOT ORIGINALLY CONCEIVED WHEN THE MOLECULE WAS DEVELOPED, AS WELL AS EARLY SIGNALS OF FOX TOXICITY SO THAT IT ALLOWS US TO FOCUS ON POTENTIAL ADVERSE EVENTS AS THE MOLECULES MOVE FURTHER ALONG DOWN THE DEVELOPMENTAL PATHWAY. A SEVENTH AREA THAT WE THOUGHT ABOUT WHICH IS SOMETHING WE HAVEN'T MENTIONED VERY MUCH HERE, AT LEAST TO MY KNOWLEDGE, IS IMAGING. THE IMAGING COMMUNITY IS OBVIOUSLY INCREDIBLY LARGE AND PROLIFIC, AND THERE'S BEEN SUBSTANTIAL INVESTMENTS MADE AND WE HAVE THOUGHT ABOUT BIOMARKERS OFTEN AS REAL BIOLOGICAL ENTITIES, BUT OPPORTUNITIES TO DEVELOP NOVEL MOLECULAR PROBES AND MOLECULAR IMAGING AND OTHER -- AND MORE STANDARD IMAGING TECHNOLOGIES IS SOMETHING THAT WE MIGHT CONSIDER, AND THIS ALSO COULD BE SOMETHING DONE IN PARTNERSHIP WITH NIBIB, SO THERE'S OPPORTUNITIES FOR SOME CROSS POLLINATION OR CROSS FERTILIZATION BETWEEN AT LEAST TWO INSTITUTES AT NIH TO DO THIS KIND OF WORK. POTENTIALLY RELATED TO THIS IS THE NOTION OF THE FACT THAT CERTAIN DEVICES AND MOBILE HEALTH TECHNOLOGIES AND CENTERS ARE BEING DEVELOPED OR MAYBE EVEN BEING DEVELOPED AND ALSO NOW MARKETED BY COMPANIES, I.T. HIGH-TECH COMPANIES, AND I GUESS THE QUESTION WE THOUGHT ABOUT IS WHETHER WE COULD ENGAGE WITH EITHER OF THOSE COMPANIES DIRECTLY IN A PUBLIC-PRIVATE PARTNERSHIP MODALITY, OR WITH SOME OF THE ACADEMIC INSTITUTIONS AS SCHOOLS OF ENGINEERING ARE ALSO COMMITTED TO SENSOR TECHNOLOGY TO BEGIN TO EXPLORE THEIR USE TO ENABLE US SO MONITOR PATIENTS IN CLINICAL TRIALS, TO THINK ABOUT DIFFERENT TYPES OF OUTCOMES THAT THESE SENSORS MIGHT ALLOW US TO DO AND POTENTIALLY DEVELOP NEW PATHWAYS A AND MEASURES FOR THERAPEUTIC DEVELOPMENT. THEN THE LAST AREA, SOMEWHAT RELAT TO THE ONE I JUST SPOKE OF, BUT THE NOTION THAT POINT OF CARE DIAGNOSTICS WOULD SEEM TO BE POTENTIALLY VERY ENABLING AND BREAKTHROUGH IN CERTAIN AREAS OF MEDICINE. THE OPPORTUNITY FOR EITHER WITHIN A PRIMARY CARE PHYSICIAN AS OFFICPHYSICIAN'S OFFICE OR EVEN IN A PATIENT'S HOME, TO BE ABLE TO SELF-MONITOR DISEASE OR HEALTH STATES WOULD SEEMINGLY BE VERY ENABLING, AND OF COURSE IN AREAS WHERE THERE ARE LIMITED RESOURCES, PARTICULARLY IN THE DEVELOPING WORLD, NOT HAVING ACCESS TO THE KINDS OF REFERENCE LABS AND HIGH TECHNOLOGY INFRASTRUCTURES THAT WE HAVE HERE, PIN POINT OF CARE DIAGNOSTICS COULD BE GAME-CHANGING. SO THE IDEA HERE WAS, AGAIN, TO WORK WITH GROUPS THAT ARE FOCUSED IN THIS, DARPA BEING ONE, GATES FOUNDATION BEING ANOTHER AND PROBABLY SELF OTHERS THAT YOSEVERALOTHERS THAT YOU WOULD BE AWA RE OF TO THINK OF HOW NCATS AND C.A.N. CAN REALLY MAKE A CONTRIBUTION AND ACCELERATE THIS IMPORTANT AREA. SO THOSE WERE THE NINE AREAS THAT WE IDENTIFIED FROM THE LONG LIST, WHICH YOU HAVE IN THE APPENDIX OF THE SLIDE DECK IF YOU HAD A CHANCE TO LOOK AT IT. I THINK AT THIS POINT, WHAT WE WOULD LIKE TO DO IS OPEN ANY OF THESE AND ALL OF THESE FOR DISCUSSION. I THINK WHAT I'D LIKE TO HEAR FROM YOU IS ARE ANY OF THESE YADIDEAS PARTICULARLY IMPACTFUL, AND EQUALLY IMPORTANT, ARE ANY OF THESE IDEAS EQUALLY USELESS THAT WETIOUS DISBAND WIT WE SHOULD DISBAND WITH AND OF MOVE ON. BECAUSE WHAT WE'RE GOING TO DO FOLLOWING THIS MEETING IS -- I DON'T THINK WE'RE GOING TO HAVE A CHANCE TO OH DO SORT OF THE RANKING HERE BUT WE'LL DO THIS SHORTLY BY EMAIL, WE'RE GOING OH BE ASKING YOU TO HELP US RANK THE IDEAS AND WE'LL SELECT THREE TO FIVE OF THEM AND ALSO ENCOURAGE SEVERAL OF YOU TO JOIN US IN DRILLING DOWN AND DEVELOPING BRIEF PROJECT PLANS THAT WOULD BE TEED UP FOR THE SEPTEMBER MEETING. SO WITH THAT IN MIND, WE HAVE ABOUT 15 MINUTES BY THE AGENDA TO HAVE A DISCUSSION NOW, WHICH IS NOT AN EXTRAORDINARILY LONG TIME, BUT FREDA AND I WOULD REALLY LIKE TO HEAR YOUR THOUGHTS ON THE IDEAS THAT WE'VE TEED UP AND, AGAIN, ANYTHING THAT WE'VE LEFT OUT THAT YOU THINK IS IMPORTANT. FRANK. >> SINCE YOU LIST DARPA, YOU SHOULD LIST FDA, RIGHT? IF YOU WORK WITH PRIVATE FOUNDATIONS AND YOU LIST DARPA -- >> THE DAD FDA, CERTAINLY. >> ALL RIGHT. SO I THINK ACTUALLY, I DIDN'T FIND ANY PARTICULAR -- >> FRANK, CAN YOU SPEAK IN THE MIC? >> SORRY. I DIDN'T FIND ANY PARTICULAR POINT USELESS, TO GIVE YOU THE SHORT ANSWER. THERE ARE MANY GOOD POINTS THAT YOU'RE MAKING, BUT I THINK THEY ALSO NEED TO BE PRIORITIZED, AND I'M NOT SURE AT THIS VERY MOMENT HOW THE HIGHEST PRIORITIES SHOULD BE SET. I THINK IT NEEDS TO BE SEEN IN CONTEXT WITH THE OTHER RESPONSIBILITIES THAT SOMEHOW HAVE TO SYNERGIZE AND, AS SUCH, I THINK IT NEEDS A LITTLE BIT MORE THOUGH BUT I ALSO BELIEV THAT IF YOU GO TOWARDS CLINICAL DEVELOPMENT AND THE IMPACT TOWARDS HEALTH OUTCOMES, YOU NEED TO CONSIDER THE COLLABORATIONS PARTICULARLY WITH THE WHOLE ORGANIZATION IN THE SIGNIFICANT RING. >> FIRST I APOLOGIZE FOR NOT INCLUDING THE FDA IN SOME OF T WHICH I THINK THERE'S PROBABLY MANY OF THEM, NOT JUST THIS LAST ONE, WHERE FDA AS AN IMPORTANT STAKEHOLDER WOULD BE VALUED IN WORKING WITH US. IN TERMS OF THE PRIORITIZATION, I THINK YOU'RE ABSOLUTELY RIGHT, AT SOME LEVEL, WE HAVE TO CONTEXTIZCONTEXTUALIZE THIS WITH ANYTHING NCATS IS DOING, BUT PARTICULARLY THE C.A.N. BOARD TO THINK ABOUT WHERE, WHICH OF THESE, IF ANY OF THEM, ARE REALLY FULL PILLING THE NEED IN A TRANSLATIONAL GAP THAT IS NOT BEING ADEQUATELY ADDRESSED AND WHERE THERE'S A REAL OPPORTUNITY TO ACCELERATE THERAPEUTIC OR DIAGNOSTIC DEVELOPMENT IN WAYS THAT HAVE NOT BEEN DONE BEFORE. OF AND ALSO TO -- FRE FREDA MENTIONED WE WANT TO HAVE PROJECTS THAT HAVE A FIVE-YEAR TIME HORIZON OR LESS. IF WE CAN. AND SOME OF THESE MAY LEND THEMSELVES TO SOME INCREDIBLY SHORT TERM WINS, AND IT WOULD BE NICE TO BE ABLE TO GO BACK TO CONGRESS AND SAY LOOK WHAT WE'VE DONE WITH A MODEST AMOUNT OF FUNDING. P WE CAN GET A LITTLE BIT MORE FUNDING, WE'LL BE ABLE TO DO MORE. THAT'S GOT TO BE PART OF THE EQUATION, I THINK. >> I THINK WE TALKED A LOT ABOUT DARPA. WE SHOULD KEEP IN MIND THAT DARPA, BY LAW, CAN ONLY FUND WHAT IS DISRUPTIVE. WHAT WE JUST REVIEWED HERE HAS TO DO WITH FIXING DYSFUNCTIONALITIES THAT WE HAVE IN A TRANSLATIONAL RESEARCH ENTERPRISE WHICH WE'RE NOT GETTING YET TO THE TOTALLY DISRUPTIVE. IT DOESN'T MEAN THAT THIS IS NOT WORTH DOING. BUT I DON'T THINK IT WILL QUITE GET US TO WHERE WE WANT TO BE IF WE CONCEIVE NCATS AS BEING THE DARPA LIKE SO TO SPEAK OF NIH. SO I CAN SEE A NECESSITY OF DOING THIS THING, BUT PERHAPS ALSO WE MAY BE TOO PRESCRIPTIVE. LIKE FOR EXAMPLE, THE BIOMARKERS. WHICH IMPLIES THAT THE BIOMARKERS IS THE ANSWER TO TARGETED THEI THERAPEUTICS AND PERSONALIZED MEDICINE. I THINK SH I THIS IS A SCIENTIFIC DEBATE WE CAN'T HAVE HERE TODAY, BUT THE FACT WE'VE SPENT SO MUCH TIME, EFFORT AND MONEY SEARCHING FOR BIOMARKERS AND WE HAVE FOUND SO FEW OF THEM SUGGESTS THAT THIS IS PERHAPS -- IT MAY BE, YOU KNOW, THE WAY TO TARGET THERAPIES IN SOME CASES, BUT MAYBE THERE'S OTHER WAYS. AND YOU KNOW, RATHER THAN TELL THE INNOVATORS OF THE WORLD, FIND MORE BIOMARKERS, I WOULD RATHER TELL THEM, FIND A WAYS TO ENABLE PERSONALIZED MEDICINE. WHETHER IT IS THROUGH BIOMARKERS OR WHATEVER ELSE THEY CAN DREAM OF. AND THE EXPERIENCES AS A GUIDE, ESPECIALLY THE EXPERIENCES OF DARPA AS A GUIDE, I THINK IT WOULD SURPRISE US WITH INNOVATIVE WAYS TO TARGET THERAPIES THAT MAY NOT NECESSARILY INVOLVE BIOMARKERS. WE SHOULD LEAVE THEM FREE TO EXERCISE THEIR CREATIVITY AS THEY SEE FIT. >> THAT'S A GREAT COMMENT. AND LET ME JUST RESPOND BY SAYING THAT A LOT OF THE INFORMATION THAT WE -- THE THINGS THAT WE'VE PRESENTED TODAY WERE, AS I SAID EARLIER, CAME FROM PREVIOUS EXERCISES, INCLUDING FROM THIS BOARD WHERE WE WERE NOT -- I DON'T THINK WE WERE NECESSARILY THINKING IN THE DISRUPTIVE MODE AT THE TIME. SO YOUR POINT IS ABSOLUTELY WELL TAKEN, AND I THINK WE SHOULD MAYBE MAKE SURE THE LANGUAGE OF WHATEVER WE END UP TRYING TO DO IN THIS SPACE IS REALLY ENCOURAGING DISRUPTIVE INNOVATION OF THE TYPE YOU'RE TALKING ABOUT RATHER THAN BEING AS PRESCRIPTIVE AS THIS MAY SOUND. THEN SECONDLY, I WOULD SAY THERE IS EVERY REASON, THERE'S ABSOLUTELY EVERY REASON THAT WE SHOULD BE WORKING ON THIS WITH DARPA. I MEAN, WE SHOULD BE ACTUALLY ENGAGING THEM PROBABLY FOR SOME OF THESE IDEAS IN BEING A COLLABORATOR WITH US AND TAKING ADVANTAGE OF THE KIND OF DISRUPTIVE THINKING YOU ALLUDED TO THAT THEY HAVE ALREADY DONE IN SOME OF THIS SPACE. SO I THINK THOSE ARE GREAT POINTS. FREDA, DID YOU WANT TO MAKE AN ADDITIONAL COMMENT? >> I ACTUALLY WAS GOING TO ASK FOR KIND OF A NEXT STEP ON THIS, WHICH IS SINCE WE SAID THESE AREN'T THE ONLY NINE THAT WE GET TO VOTE ON, EXPAND THAT, IF YOU WERE DOING KIND OF AN ALTERNATIVE TO NINE AROUND HOW WOULD YOU GET TO TARGETED THERAPIES WITHOUT PURSUING BIOMARKERS AS WE RECOGNIZE THEM. CAN YOU HELP US KIND OF SHAKE THE LANGUAGE OF WHAT THIS PROJECT WOULD LOOK LIKE? BECAUSE I THINK THAT THAT'S THE KIND OF THING THAT WE COULD THEN PRPRIORITIZE, SO IT WOULD BE, YOU KNOW, KIND OF THE 9A OR THE ANTI9, I'M NOT QUITE SURE HOW TO SAY IT OF THIS, WIT WHAT THAT LANGE LANGUAGE WOULD LOOK LIKE SO WE CAN PUT IT ON OUR LITTLE BALLOT. >> I THINK DARPA HAS A GOOD WAY TO PUT IT, WHEREBY THEY WILL SAY THAT THE OBJECTIVES ARE TOP-DOWN. SO IN THIS PARTICULAR CASE, THE OBJECTIVE WOULD BE HOW DO WE ENABLE PERSONALIZED MEDICINE? THAT'S WHAT WE WANT TO DO. THE SOLUTION ON THE OTHER HAND IS BOTTOM UP. SO THE SOLUTION IS LEFT TO THE PEOPLE WHO WANT DAR A PA FUNDING DARPA FUN DING TO COME UP WITH DISRUPTIVE STUFF, DARPA CAN ONLY SPEND MONEY IF IT'S DISRUPTIVE. WE COULD EMULATE THAT HERE BY SAYING, OKAY, WE WANT TO ENABLE PERSONALIZED MEDICINE. WE'RE NOT GOING TO TELL YOU HOW TO DO IT BECAUSE WE'RE NOT SMART ENOUGH, YOU GUYS FIGURE IT OUT, AND YOU COME BACK TO US, AND IF YOU WANT SOME OF OUR MONEY, YOU'D BETTER BE DISRUPTIVE. >> WE COULD ALSO NOT JUST LIMIT THE DISCUSSION TO PERSONALIZED MEDICINE. I HAPPEN TO LIKE THAT TOPIC MYSELF, BUT WE COULD ALSO SAY LET'S FIND DISRUPTIVE TECHNOLOGIES TO ENHANCE PATIENT RECRUITMENT. I MEAN, SOME OF THE KEY GAPS THAT WE HAVE IDENTIFIED, WE SHOULD TRY TO ENGAGE IN GETTING MU IDEAS THAT HAV -- NEW IDEAS THAT HAVE NEVER BEEN THOUGHT OF BEFORE AND OBVIOUSLY BY DEFINITION, THEY'RE GOING TO BE HIGH RISK. >> CAN I JUMP IN AND JUST SAY THAT IN A PRELIMINARY SENSE, NUMBER FIVE AND NUMBER NINE, FROM THE GIVEN LIST, ALTHOUGH I LIKE THE ANTI-9 APPROACH, 5 AND 9 ARE VERY INTERESTING TO ME, AND I WOULD PREDICATE MY SUPPORT FOR NUMBER 5 ON A QUESTION THAT -- I DON'T KNOW IFFY LANE IS JUST WALKING BACK IN OR MAYBE PETRA WILL LEARN THIS, BUT MY QUESTION HAS TO DO WITH THE FOLKS THAT HAVE COME THROUGH THE KL2 PROGRAMS, WHO ARE CHILDREN IN SOME SENSE, OR WE SHOULD TAKE SOME RESPONSIBILITY FOR THEM. I'D LIKE TO KNOW HOW THEY'RE DOING, IF WE STILL TALK TO THE K TO R TRANSITION, OR IN THEIR CAREER DEVELOPMENT, AND WOULD THE MICRO AWARDS HELP SOME OF THEM, ARE THERE SOME, YOU KNOW, MEMBERS OF THAT COHORT WHO ARE ON THE VERGE, AND WE JUST NEED TO DOUBLE DOWN A LITTLE BIT ON THEM TO HELP THEM DISRUPT OR CREATE. SO THAT'S SOMETHING MAYBE AT A FUTURE COUNCIL MEETING, WE COULD HEAR ABOUT HOW THOSE FOLKS ARE DOING AND SEE HOW WE CAN HELP THEM. >> I DON'T HAVE ANY DATA ON THE TOP OF MY HEAD, BUT I KNOW THAT THESE THINGS ARE BEING TRACKED AND THIS IS A VERY IMPORTANT AND CONTINUING PROBLEM THAT THIS TRANSITION IS DIFFICULT, SO I WOULD IMAGINE THAT THIS MIGHT BE VERY HELPFUL. TO REALLY CAPITALIZE OR MAXIMIZE THE RETURN ON THAT INVESTMENT. BECAUSE IF THEY -- BUT THEN NEED YEARS TO GET SOMEWHERE AND IF YOU KNOW THE AGE OF THE -- IS RATHER HIGH, THAT WOULD BE AN ISSUE AND A LOSS OF REALLY GREAT POTENTIAL AND TIME. >> I'D LIKE TO COMMENT ON TWO ASPECTS. FIRST OF ALL, ON POE CUSS NUMBER 2, I THINK THE CRITICAL ISSUE IS PREDICTIVE TOXICOLOGY. THERE ARE SOME VERY GOOD PROGRAMS GOING ON, RIGHT NOW, FOR EXAMPLE, THE DRUG INDUCED LIVER INJURY NETWORK, PAUL WATKINS' EFFORT. I THINK PARTLY FUNDED BY THE NIH, PARTLY SUPPORED BY THE INDUSTRY AND PARTLY SUPPORTED BY PRIVATE FOUNDATIONS. THAT KIND COULD HAVE EASILY GONE A LONG WAY TOWARDS LIVER INDUCED DRUG INJURY, BUT OF COURSE THERE ARE OTHER ELEMENTS OF DRUG INJURY INCLUDING CARDIOVASCULAR INJURY, SKIN ISSUES, THESE ARE THE THINGS THAT KILL PRODUCTS. SO THIS IS THE KIND OF THING THAT ONE DRUG COMPANY CAN'T DO, INFORMATION FROM MULTIPLE DRUG COMPANIES OR THE WHOLE INDUSTRY CAN BE VERY VALUABLE IN DEFINING THE MODELS THAT WILL HELP LEAD TO PREDICTED TOXICOLOGY. >> COULD I REQUEST ASK A CLARIFYING QUESTION ABOUT THAT? DO YOU VIEW THIS AS A PRE-COMPETITIVE SPACE THAT IN PRINCIPLE INDUSTRY SHOULD BE WILLING TO JUST SHARE AS MUCH OF THE INFORMATION WITH ONE ANOTHER THAT -- >> ABSOLUTELY. AND IN FACT, THAT'S WHAT'S HAPPENING RIGHT NOW, WITH THE SO-CALLED -- NETWORK THAT PAUL WATKINS HAS SET UP AT THE UNIVERSITY OF NORTH CAROLINA. I THINK THERE ARE A DOZEN COMPANIES INVOLVED IN THAT. NIH SUPPORT AND ALSO SUPPORT -- SOME GREAT INTEREST FROM THE FDA SPECIFICALLY IN THIS. SO I THINK IT'S A VERY IMPORTANT AREA, BECAUSE A LARGE PART OF GETTING DRUGS TO PATIENTS IS OVERCOMING THE TOXICITY WHICH EVERY DRUG HAS. THE OTHER COMMENT I WANT TO MAKE IS ON GENERAL FOCUS NUMBER 5. YOU HAVE THIS AS A MICRO AWARD TO GET PAST SOME SMALL PRECLINICAL HURDLE. BUT I THINK IT COULD BE MICRO AWARDS TO TEST VERY INOWE VAI POTENTIALLY DISRUPTIVE IDEAS. THIS IS A PROGRAM THAT I WAS ABLE TO SET UP WITH THE GATES FOUNDATION THROUGH WHAT WAS CALLED GRAND CHALLENGES, EXPLORATIONS, $100,000 GRANT TO TEST TRULY CREATIVE IDEAS, NO PRELIMINARY DATA REQUIRED, VERY SMALL, TWO-PAGE APPLIC AND A REVIEW PROCES WHICH WAS SPECIFICALLY NOT PEERS, BECAUSE THE CONCEPT WAS THAT PEERS DON'T -- INOWE VAI T INNOVATORS DON'T HAVE PEERS, THEREFORE, YOU SHOULD NOT HAVE PEERS REVIEW THE GRANT. >> RIGHT. >> AND THAT PROGRAM HAS BEEN HUGELY SUCCESSFUL AT THE GATES FOUNDATION, I CAN TELL YOU THAT WE ANTICIPATED THAT MAYBE TWO OR 3% OF THESE GRANTS WOULD BE ELIGIBLE FOR THE NEXT ROUND, WHICH WAS A MILLION DOLLARS IF THEY REALLY SHOWED PROOF OF SEPTEMBER FOR TRULY NOVEL -- THE HIT RATE HAS BEEN OVER 10%, WHICH IS BEYOND OUR WILDEST DREAMS. OF COURSE MANY OF THESE IDEAS ARE MOVING FORWARD. SO NOT SO THIS PROGRAM, I TRULY BELIEVE IN, BUT IT SHOULD BE FOCUSED ON INNOVATION RATHER THAN -- >> OR BOTH. >> I'D LIKE TO JOIN IN THE COURSCHORUS OF SUPPORT ABOUT 5 AND MAKE TWO QUICK COMMENTS. ONE IS A SIDE BENEFIT IS IT'S AN ASSAY OF WHAT'S INTERESTING THAT'S GOING ON OUT THERE THAT THAT HELPS US EDUCATE OURSELVES AT NCATS. A CAVEAT IS YOU HAVE TO HAVE A REALLY GOOD REVIEW PANEL, AND IT'S NOT YOUR REGULAR REVIEW PANEL TO DO THIS. >> THANKS. WE'RE FAIRLY CLOSE TO THE END OF THE SESSION, BUT I WOULD LIKE TO MAKE SURE EVERYBODY WHO HAS SOMETHING TO SAY HAS A CHANCE. ARE THERE ANY OTHER COMMENTS ON THESE PROJECTS OR ANY OTHER IDEAS THAT YOU'D LIKE TO BRING FORWARD AT THIS TIME? IF NOT -- FIRST OF ALL, THANK YOU ALL FOR IN A VERY SHORT PERIOD OF TIME, YOU KNOW, PUTTING SOME FOCUS ON SOME OF THE KEY AREAS THAT WE WILL TAKE GOING FORWARD, AND I'VE HEARD THE TERM DISRUPTIVE SEVERAL TIMES NOW, AND ALSO THE NOTION OF ENGAGING OTHER DISRUPTIVE ORIENTED GROUPS SUCH AS DARPA. WE CAP YOU'RED A LO CAPTURED A LOT OF YOUR IDEAS. WE'LL TWEAK THE LANGUAGE TO MAKE SURE THAT WE'RE REFLECTING EVERYTHING YOU SAID IN THIS SHORT PERIOD OF TIME. WE'RE GOING TO SEND OUT -- WE HAVEN'T REALLY DEVISED THE PROCESS EXACTLY, BUT WE'RE GOING TO SEND THIS OUT TO ALL THE C.A.N. BOARD MEMBERS WITH A RANKING LIST THAT WILL ASK YOU TO FILL OUT, MAKE ANY COMMENTS THAT YOU HAVE, ALSO THERE'S AN OPPORTUNITY THERE TO ADD SOME OTHER IDEAS THAT YOU MAY HAVE THOUGHT OF AFTER THIS SESSION. THEN WE'RE GOING OH ASK PORE SOME VOLUNTEERS. I DON'T THINK THIS IS GOING TO BE A LARGE AMOUNT OF HEAVY LIFTING, BUT WE REALLY WANT TO CREATE A SERIES OF ONE, POTENTIALLY TWO PAGE DOCUMENTS THAT DRILL DOWN ON WHAT THE OPPORTUNITIES ARE AND SHORT TERM/LONG TERM POTENTIAL DELIVERABLES THAT WE MIGHT SEEK FROM FUNDING SO THAT WE CAN HAVE A ROUNDER DISCUSSION, MORE IN DEPTH DISCUSSION AT THE SEPTEMBER MEETING. SO WHEN WE ASK FOR YOUR RANKINGS, WE'LL ALSO ASK IF YOU'RE WILLING IT TO PARTICIPATE IF ONE OF THOSE PROJECTS WERE SELECTED FOR -- AS A TOP PRIORITY TO HELP US CRAFT IT IN A WAY THAT'S GOING TO BE APPETIZING BOTH TO C.A.N. AND NCATS AS WELL AS TO DEVELOP THE FUNDING VEHICLES THAT WE WOULD LIKE TO SEE ELICIT IF WE GET THE $29 MILLION APPROPRIATION. THANK YOU. >> EXCELLENT. THANK YOU, GEOFF, THANK YOU, FREDA, AND THANK YOU, DAN. WE ARE GOING TO MOVE OF ON TO THE NEXT AGENDA ITEM, WHICH IS THE REPORT FROM THE COUNCIL SUBCOMMITTEES. SO SINCE THE LAST MEETING, EACH OF THE GROUPS HAS DONE A LOT OF WORK IN THESE AREAS, AND SO I JUST WANT TO EMPHASIZE WHAT WE MEAN BY REPORT. A COUNCIL SUBCOMMITTEE IS SET UP TO BE -- IF AN INSTITUTE THINKS THAT THERE IS AN ISSUE WHICH IS GOING TO BE OF ONGOING IMPORTANCE TO THE INSTITUTE OVER TIME, AND -- [PLEASE STAND BY] >> IS THIS GEORGE? OR FRANK? >> FRANK IS ON LINE. >> WHO WAS SPEAKING EARLIER? >> NO, I'M NOT ON THE AIR. I'VE BEEN QUITE QUIET ON MY END. [LAUGHTER] >> SORRY FOR THAT INTERRUPTION. I HAD BEEN FIGURING OUT HOW TO PUT MYSELF ON AND OFF MUTE, SO I WILL GO BACK ON MUTE. >> THANK YOU, BOTH GEORGE AND FRANK, FOR JOINING US. WE'RE ABOUT TO SPHART THE SUBCOMMITTEE PART OF THE AGENDA. >> SO WHEN WE SAY REPORT, THIS IS DIFFERENT FROM THE WORKING GROUP REPORT SO THE WORKING GROUP REPORT GOES OUT, THEY SPEND SIX MONTHS FIVE MONTHS IN THIS CASE, THEY COME UP WITH A PACKAGE, THEY DELIVER THE PACKAGE AND THE WORKING GROUP IS OVER AND THEN WE TAKE THAT AS A COUNCIL AND AS A CENTER AND MOVE FORWARD. SUBCOMMITTEE REPORTS ARE MORE SORT OF IN THESE DIFFICULT AREAS THAT WE'RE NOT GOING TO SOLVE IN FIVE MONTHS, WHAT IS YOUR CURRENT THINKING? AND SINCE THE LAST TIME? AND THESE ARE MORE SORT OF UPDATES, I GUESS IS A BETTER WAY TO PUT IT, THAN A REPORT. A REPORT HAS A SENSE OF FINALITY TO IT. THIS IS MORE OF AN UPDATE. AND THAT'S TO SAY THAT EACH OF OUR THREE SUBCOMMITTEES, YOU'RE NOT GOING TO BE ALLOWED TO GO AWAY, JUST BECAUSE YOU ISSUE A REPORT THIS TIME, I WOULDN'T ASK Y TO DO THE SAME THING AT MANY COUNCIL MEETINGS IF NOT ALL OF THEM, NOT ONLY TO COME UP WITH NEW IDEAS, BUT TO HELP US WITH -- IN THE OTHER PROGRAMS THAT NCATS ARE DOING, IF YOU CAN LOOK AT THOSE PROGRAMS THROUGH THE LENS OF THE SUBCOMEE THAT YOU'RE ON, AND TELL US HOW WE'RE DOING, AND WHETHER WE SHOULD CHANGE SOME OF THESE PROGRAMS TO BE BETTER ONE WAY OR ANOTHER, THAT WOULD BE REALLY HELPFUL AS WELL. MARGARET AND MYRL, DO YOU WANT TO START OUT? >> MYRL MAYBE I'LL START. OF WE HAVE THREE SLIDES WHICH BASICALLY CAPTURE SOME OF THE WORK WE'VE BEEN DOING. IF YOU WANT TO PUT UP THE FIRST SLIDE. SO THE PATIENT ENGAGEMENT GROUP RIGHT NOW CONSISTS OF MYRL AND MYSELF, BOB BELL, LOU DE JANEIRO -- AND SCOTT WE'RE. WE KNOW WE'VE GOT THE MANDATE TO EXPAND THAT BUT WE REALLY FELT LIKE LET'S KEEP IT TIGHT AND FOCUSED BEFORE WE ADD TO THAT MIX. I HAVE TO SAY, AND I THINK I MENTIONED THIS BRIEFLY WHEN WE WERE PRESENTING THE LAST TIME THAT WE'VE STRUGGLED A BIT BUT I FEEL LIKE WE'VE REACHED SOME AREAS THAT ARE LOW HANGING FRUIT OPPORTUNITIES, AND THEN IF WE ARE IN THIS FOR THE REST OF OUR LIFE, WE'LL GET TO THE BIGGER STUFF LATER. SO THE SHORT TERM GOAL IS OH TO DEVELOP THIS MASTER FRAMEWORK FOR PATIENT INVOLVEMENT IN NCATS PROGRAMS AND PROJECTS THAT ALL THE DIFFERENT STAKEHOLDERS CAN OWN. SO IF YOU LOOK AT THIS SLIDE, YOU'VE GOT NCATS, YOU'VE GOT DIFFERENT PATIENT DISEASE RESEARCH ORGANIZATIONS, YOU'VE GOT THE THEMES OF PATIENT ENGAGEMENT, WHICH I HAVE TO SAY RIGHT NOW, THAT IS A HUGE BUZZ WARD, A AND BERNARD, YOU WERE TALKING ABOUT DISRUPTIVE INNOV TO ME FEELS A LITTLE SIMILAR TO THAT, BUT YOU CAN'T BUMP INTO SOMETHING RIGHT NOW WITHOUT SOMEBODY CALLING IT PATIENT ENGAGEMENT. THE PHARMACEUTICAL INDUSTRY HAS REALIZED THAT THAT'S A GOOD WAY TO GO. T BIOTECH INDUSTRY, I MEAN, SOME COMPANIES ARE CREATING POSITIONS THAT THIS IS THEIR WHOLE SOLE FUNCTION, SO WE HAVE TO BE CAREFUL OF FALLING INTO SOME TRAPS, I THINK, AROUND THAT. AND HENRILY LOOKIN HEN THEN REALLY LOOKING AT PAST PRACTICES. SO MYRL, I'LL PASS IT TO YOU IF YOU WANT TO GO THROUGH SOME OF THESE MACRO LEVEL AREAS HA YOU HELPED US TO STRUCTURE. >> THERE ARE THREE MAJOR POINTS THAT WE KIND OF CAME UP WITH. IT'S INTERESTING TO ME, HAVING HEARD THE REPORT THAT WAS IN RESPONSE TO THE IOM REPORT, THE APPROACH IS FAIRLY SIMILAR IN THAT WE WANT TO BE QUITE SOPHISTICATED, ET CETERA. SO THE FIRST THING WE SAID WAS, AS A SUBCOMMITTEE, WE NEEDED MORE BACKGROUND, A LOT MORE BACKGROUND, EVEN TO DO WHAT CHRIS JUST DESCRIBED, WE NEED TO KNOW MORE, AND SO ONE OF THE SUBTHINGS UNDER IT, AND I'LL JUST GIVE SOME EXAMPLE, WAS THAT WE NEEDED TO DETERMINE HOW AND IN WHAT CONTEXT PATIENT ENGAGEMENT IS CURRENTLY HAPPENING IN ALL OF THE DIFFERENT PROGRAMS IN NCATS, SO WE HEARD A LOT ABOUT THE CTSAs THIS MORNING, ALTHOUGH STILL NOT AT THE LEVEL WE WOULD NEED TO KNOW IS IT WORKING OR NOT, TREND, ORDR AND OTHERS. WE ALSO HAD JUST BEGUN TO LOOK AT SOME OF THE OTHER NIH ENTITIES. AND ONE OF THE THINGS THAT WE HAVE SOME INFORMATION ABOUT IS NCI AZ OFFIC'S OFFICE OF ADVOCATE IS SEE RELATIONS, WANTED TO KNOW WHAT ARE THEY DOING, HOW ARE THEY DOING IT, WHAT'S WORKING, ET CETERA. THE SECOND BIG BUCKET WAS IDENTIFYING THE OPPORTUNITIES FOR WHAT WE WOULD CALL MEANINGFUL PATIENT ENGAGE. , AND WE PROBABLY NEED TO DEFINE THAT. SO THERE WE TALKED ABOUT WORKING WITH NCATS STAFF TO REALLY CODIFY THE OPPORTUNITIES THAT WE ARE AWARE OF RIGHT NOW THAT WOULD BE BASED ON SOME OBJECTIVES WE WANT TO ACCOMPLISH, SO WHY, WHAT ARE THE OBJECTIVES. WE THINK IT'S REALLY IMPORTANT TO DEVELOP THOSE GOALS, THEN DEVELOP GUIDANCE DOCUMENTS FOR ALL OF THE NCATS ENTITIES AND PROGRAMS SO THAT WE CAN -- THEY CAN IDENTIFY THEIR OWN OPPORTUNITIES FOR PATIENT ENGAGEMENT, THEY SET OBJECTIVES BASED ON THEIR PROGRAM AND WHAT THEY'RE TRYING TO ACCOMPLISH AS THEY ENGAGE PATIENTS, GUIDANCE FOR HOW TO RECRUIT APPROPRIATE PATIENT ENGAGEMENT, PATIENT PARTICIPANTS, AND THEN TO REALLY IDENTIFY WHAT THE TRAINING NEEDS ARE AND STRATEGIES TO MEET THOSE NEEDS. SO THAT WE CAN TRAIN PEOPLE TO THE CORE COMPETENCIES THAT ARE NEEDED FOR THAT TASK TO MEET THOSE OBJECTIVES. THEN ONE OF THE THINGS WE NOTED BECAUSE CHRIS SAYS IT, EVERYTHING IN NCATS HAS A PARTNER. SO I'M CLEAR AT THIS POINT OF IF NCATS SAYS WE HAVE TO ENGAGE PATIENTS, WHAT IS A PARTNER, AGREEMENT ON THE NEED, THE IMPORTANCE OF ENGAGING PATIENTS AND HOW THEY'LL DO IT, SO WE THOUGHT IT WOULD BE GREAT TO OVER TIME DEVELOP SOME STRATEGIES FOR HOW TO ACCOMPLISH THAT TOGETHER. AND THEN LASTLY, THE THIRD BIG BUDGET, I'M NOT FOLLOWING THIS EXACTLY, BUT I'M GETTING THERE -- IS TO IDENTIFY AND CON TO REFINE WHATEVER BEST PRACTICES THAT WE IDENTI AS WE OF MOVE ALONG. ONE OF THE THINGS AS I SAID, SETTING OBJECTIVES, WHICH WE HEARD ABOUT EARLIER TODAY, MEASURABLE OBJECTIVES, BUT DETERMINE WHAT THE DATA IS THAT ESPECIALLY CHRIS ULTIMATELY WOULD WANT TO COLLECT TO KNOW ARE THE OBJECTIVES THAT WERE SET FOR THE PATIENT ENGAGEMENT BEING MET, WHAT'S WORKING AND WHAT ISN'T, IDENTIFY WHAT MEASURES THAT WE WOULD WANT TO MONITOR AROUND PATIENT ENGAGEMENT, BOTH SOME MAY BE SPECIFIC TO PROGRAMS BUT SOME ACROSS ALL OF NCATS. THEN THAT WOULD ALLOW US TO IDENTIFY THE GAPS ON THAT NEXT TO THE LAST, SO WE COULD REALLY LOOK AT WHAT ARE THE GAPS AND WHAT ARE THE NEEDS THAT JUST DON'T EXIST RIGHT NOW. AND THEN FINALLY, SPHEAB POLICY AND PROCEDURES FOR SHARING PATIENT ENGAGEMENT STRATEGIES, ALL THE BEST PRACTICES ACROSS ALL THOSE DIFFERENT TYPES OF ACTIVITIES I MENTIONED, SO THAT THEY COULD BE EFFECTIVELY IMPLEMENTED THROUGHOUT NCATS. AND I AM ALL FOR DISRUPTIVE SOLUTIONS TO ALL OF THESE ISSUES. THAT'S IT. THANK YOU. >> NEXT SLIDE S. SO IF YOU TAKE THE BUCKETS THAT MYRL JUST PUT OUT THERE, THEN TRANSLATE IT BACK INTO WHAT ARE SOME REAL OPPORTUNITIES TODAY, ONE OF THEM THAT'S NOT UP HERE BUT CHRIS YOU MENTIONED IT THIS MORNING, YOU TALKED ABOUT DOING AN OVERHAUL OF THE WEBSITE. SO AS JUST A LITTLE PILOT, I ASKED THE TEAM AS AN ASSIGNMENT, I SAID TAKE 20, 30 MINUTES, GO ON THE NCATS WEBSITE, AND FISH AROUND WITH PATIENT ENGAGEMENT AS YOUR SORT OF MINDSET, AND I THINK EVERYBODY FELT THAT THERE ARE MORE OPPORTUNITIES TO HIGHLIGHT PATIENT ENGAGEMENT THAT'S ALREADY EVEN HAPPENING WITHIN THE NCATS PORTFOLIO ON THE WEBSITE. SO I THINK THE TIMING IS GOOD IN TERMS OF YOU TAKING A LOOK AT IT. EVEN JUST NOW I WAS FISHING AROUND ON SOME OF THE OTHER INSTITUTES' WEBSITES, NCI, NINDS, NIAID. IT'S NOT THAT PATIENTS -- ADMITTEDLY THEY HAVE A EASIER TASK BECAUSE THEY HAVE ONE THING THAT THEY'RE SUPPOSED TO BE FOCUSING ON, SO IF YOU LOOK AT NCI, RIGHT ON THE HOME PAGE, THERE'S PROBABLY VERY CONSUMER-PATIENT-FRIENDLY INFORMATION ABOUT A MULTITUDE OF CANCERS. SO IF YOU'RE OUT THERE, YOU KNOW, IN GOOGLE HELL AND ALL OF A SUDDEN YOU SAY, OH, MY GOSH, I WANT TO KNOW ABOUT THIS CANCER, YOU'RE GOING TO HIT THAT RIGHT AWAY. WELL, THE LIKELIHOOD OF A PATIENT OUT IN THE WORLD SAYING, AH RVETION I NEED THIS ONE KEY TERM OUT OF TRANSLATIONAL RESEARCH AND NCATS IS THE PLACE FOR ME IS PROBABLY NOT GOING TO BE THE SAME, BUT IF BACK TO WHAT RON WAS TALKING ABOUT EARLIER, THIS IDEA THAT THE PATIENT ORGANIZATION IS CHANGING AND DISRUPTING BIOMEDICAL RESEARCH, THEN WE WANT TO MAKE SURE THAT WE'RE CAPTURING THAT ENERGY AND THAT ENVIRONMENT ON THE NCATS WEBSITE BECAUSE YOU DO HAVE REAL TANGIBLE EXAMPLES WHERE YOU'VE ALREADY BEEN DOING THESE COLLABORATIONS AND THEY'RE MAKING A DIFFERENCE. I THINK IT ALSO GOES BACK TO ANOTHER THEME WE'VE TALKED ABOUT BEFORE WHICH IS WHAT'S THE STORY LINE OF NCATS, SO THAT THIS PATIENT ENGAGEMENT PIECE DOESN'T FEEL LIKE AN OUTLIER, IT BECOMES PART OF THE EXISTING DNA OF NCATS. I THINK IF YOU'RE DOING AN ANALYSIS OF ALL OF THE NCATS PROGRAMS AND STARTING TO FIGURE OUT HOW DO YOU ENGAGE WITH PATIENTS, THEN SOME OF THE LOW HANGING FRUIT COULD BE ENGAGING PATIENTS SOONER IN THE RESEARCH PROCESS. SO CAN WE, OVER TIME, LOOK AT THE PEER REVIEW PROCESS? ONE OF THE FOLKS IN OUR GROUP SAID THAT THE IDEA OF INFUSING PATIENT PERSPECTIVE SOONER IN THE PROCESS AND NOT JUST AN END OF THE PROCESS IS SOMETHING THAT MANY OTHER INSTITUTES AND EVEN DOD, THEY HAVE EXAMPLES THAT YOU CAN LOOK A AT SO YOU DON'T HAVE TO START ALL THIS FROM SCRATCH. I THINK THAT'S SOMETHING YOU JUST NEED TO BE CONSCIOUS OF, THAT IT'S NOT JUST AD HOC BUT THERE IS SOME SORT OF METHOD TO DOING THIS. THE S THING IS CREATING AN ON RAMP, AND I THINK THAT'S UP THERE, SO CAN YOU CREATE A PART OF THE WEBSITE THAT'S SORT OF AN ON RAMP TO IF YOU'RE A DISEASE FOUNDATION, YOU KNOW, HERE'S 10 THINGS EITHER WE'VE GOT FOR YOU OR 10 RESOURCES YOU SHOULD KNOW ABOUT, AND THERE'S SOME INTERRELATIONSHIPS WITH OTHER THINGS UP HERE. ONE OF THE THEMES WE HEARD WAS TO TAKE THE BODY OF KNOWLEDGE THAT EXISTS OUT THERE AMONG SOME OF THE LARGER DISEASE FOUNDATIONS AND BE ABLE TO KIND OF EACH ONE TEACH ONE, SO PARTICULARLY FOR SMALLER DISEASE FOUNDATIONS THAT ARE JUST GETTING GOING, IS THERE A ROLE FOR NCATS WILL, JUST EVEN IN TERMS OF A TWO-PAGE FACT SHEET. THIS IS SOMETHING THAT I DON'T KNOW WHAT YOUR HIRING RULES ARE BUT AN INTERN, A TEAM OF INTERNS COULD CRAFT THIS STUFF IN A MONTH OR TWO. SO THAT IT'S NOT -- IT DOESN'T HAVE OH B TO BE A HEAVY ORGANIZATIONAL LIFT. THEN LASTLY, I'D SAY THE CTSA PIECE THAT WE HEARD ABOUT EARLIER TODAY, THERE IS SUCH AN AMAZING AMOUNT OF OPPORTUNITY THERE TO ENGAGE THE PATIENT VOICE, THE PATIENT COMMUNITY ORGANIZATIONS, UNDER THE BUCKET OF COMMUNITY, AND I THINK THAT'S THE PIECE THAT MYRL AND I FELT LIKE WE WERE JUST SCRAPING THE SURFA IN TERMS OF OUR UNDERSTANDING OF WHAT'S ALREADY OUT THERE AND THEN WHAT'S TO COME. SO I THINK THE ALIGNMENT OF ALL OF THIS IS SOMETHING THAT FROM NCATS, YOU GUYS ARE GOING TO HAVE TO HELP US THREAD IT TOGETHER, BECAUSE MAYBE THAT WAS PART OF OUR CHALLENGE AS WE WERE FEELING A LITTLE BIT LIKE WE WERE WAY -- A SATELLITE WAY, WAY FAR AWAY FROM THE MOTHER SHIP IN TRYING TO KIND OF FIGURE OUT HOW DO WE GET BACK. WE THINK IT WORKED OUT WELL IN THE MOVIE "GRAVITY," BUT I HEAR THERE'S A PART 2 WHERE SHE'S ON EARTH AND THERE'S NOBODY ELSE THERE, SO WE'LL SEE. [LAUGHTER] >> SO THAT'S OUR REPORT. AND I KNOW WE WERE DRIVING YOUR STAFF CRAZY BECAUSE WE DIDN'T GET OUR SLIDES DONE, BUT BETWEEN ALL OF OUR SCHEDULES, YOU GIVE BUSY PATIENT REPRESENTATIVES THESE JOBS AND THEY GET IT DONE BUT IT'S NOT ALWAYS ON YOUR TIMELINE. >> AS YOU KNOW, WE'RE FLEXIBLE AND ADAPTIVE, AND SO WE APPRECIATE THAT. REALLY INTERESTING THOUGHTS. AND THIS IS THE KIND OF THING THAT'S ACTUALLY VERY -- ACTUALLY IT'S VERY USEFUL FOR US TO HEAR. AND I THINK AS WE MOVE FORWARD PARTICULARLY WITH THE CTSA WORKING GROUP REPORT AND HOW TO IMPLEMENT THAT, IT WOULD REALLY BE IMPORTANT FOR US TO BE ABLE TO UTILIZE YOUR KNOWLEDGE AND HOW MUCH YOU'VE THOUGHT ABOUT THIS. I THINK ONE OF THE THINGS THAT IS A CONUNDRUM FOR ME IS REALLY THE ONE AT THE TOP THERE, HOW DO WE DO THAT SOON IN THE PROCESS. AND TO HAVE IT BE A ROUTINE PART OF WHAT WE DO, NOT -- WE JUST WOULDN'T GO OUT ONCE AND KIND OF DO A LANDSCAPING OR WHATEVER, WE CAN DO THAT, BUT HOW DO WE MAKE IT PART OF A -- YOU USED THE WORD PROCESS THERE. THAT'S REALLY A MAJOR ISSUE. MAYBE WE'LL GET A PRESENTATION AT SOME POINT ABOUT THE WEBSITE. WE'RE VERY MUCH THINKING ABOUT THE WEBSITE IN THIS WAY. IT WAS REALLY CLEAR TO US THE MORE WE LOOKED AT IT, IT'S REALLY A -- IT'S NOT A WEBSITE THAT IS TERRIBLY INVITING NOW, SO WE WANT TO CHANGE -- THAT IS A BIG CHANGE. >> ONE THAT I WAS LOOKING AT, AND I CERTAINLY WAS FAMILIAR WITH IS TOM INSEL, THE NIMH SITE. THAT'S A BODY OF WORK THAT OBVIOUSLY HAS SUCH CONNECTIVITY TO A PATIENT COMMUNITY THAT NEEDS IT, AND THEY EVEN IN TERMS OF THE GRAPHICS THAT THEY USE, THE APPROACH THAT THEY'RE TAKING IN IMPARTING INFORMATION, IT INVITES YOU IN. SO IT'S AMAZING WHAT EVEN A FONT CAN DO. >> JUST A COUPLE QUICK FOLLOW-UPS. ON THE LAST POINT, PEOPLE FROM THE PLAIN LANGUAGE FIELD, PFIZER ESPECIALLY, I KNOW WE'VE RECOMMENDED TO OTHER COMPANIES AND THEY'VE USED PEOPLE THAT WE HAPPEN TO KNOW THERE'S A CADRE OF INDIVIDUALS AND THEY ARE ABSOLUTELY EXPERT AT THE LANGUAGE, AT THE SPACING, AT THE CULTURAL RELEVANCY, EVERYTHING. SO THEY'RE EXPERTS HA KNOW HOW TO DO THIS, AND THEY'VE DONE IT ACROSS MANY OTHER FIELDS AND NOT JUST RESEARCH. ON THE OTHER POINT ABOUT EARLY IN THE PROCESS, I THINK IT'S JUST IDENTIFYING, AND WE COULD HELP WITH THAT, I'M SURE, OUR GROUP, WHERE THIS IS ALREADY BEING DONE. I MEAN, SOME COMPANIES, AND MT ALLUDED TO IT IN OUR MEMBERSHIP, THEY'RE IN THIS PROCESS OR ALREADY DOING IT. I KNOW PFIZER IS REALLY LOOKING AT THIS. SANIFI, WHERE IS WHERE MARGARET -- ON OUR STAFF WAS JUST AT A MEETING IN FRANCE AND THEY'RE LOOKING AT FROM THE GET-GO, I'VE BEEN SOMEWHAT INVOLVED, SO I WANTED TO GIVE ONE EXAMPLE OF HOW TO THINK ABOUT THIS. THERE WERE RESEARCHERS WORKING ON PSORIASIS, SO YOU HAVE THE OUTBREAKS ON YOUR SKIN, AND THEY FOCUSED ALL THEIR RESEARCH ON MAKING THOSE LESION-TYPE PLACES S LATER, THEY ASKED PEOPLE WITH THE CONDITION, WHAT ARE THE OUTCOMES YOU WANT, WHAT WOULD BE IMPORTANT? THEY DIDN'T CARE ABOUT THAT AT ALL. I DO NOT WANT IT ON MY FACE BECAUSE I CAN'T GO OUT AND BE SOCIAL, AND I DO NOT WANT IT ON MY JOINT BECAUSE THAT'S WHERE IT'S PAINFUL. NOBODY ASKED. SO AT THE VERY BEGINNING, WHEN YOU EVEN THINK ABOUT WHAT ARE THE QUESTIONS THAT ARE MOST IMPORTANT RELATED TO THESE CONDITIONS OR PROCESSES, AS LONG AS YOU FIND THE RIGHT PEOPLE AND THEY'RE TRAINED FOR THE TASK, THAT'S THE KIND OF VALUE THEY CAN BRING FROM THE GET-GO. >> SO ONE OF THE THINGS THIS REALLY HITS ME WITH IS THERE'S SOME ORGANIZATIONS THAT ARE WORKING WITH THIS. THE FDA HAS FIELDED PATIEN CENTERED DRUG DEVELOPMENT, PCORI IS WORKING ON THIS, INDIVIDUAL COMPANIES HAVE KIND OF NAMED PATIENT FOCUSED INDIVIDUALS, DEPARTMENTS, AND OTHERS THAT ARE INVOLVED IN THIS WORK. WE COULD GO ON AND ON WITH THE NUMBER OF ORGANIZATIONS THAT ARE ACTIVELY INVOLVED, EVEN IF YOU JUST TOOK THE TOP CIRCLE ON THIS SLIDE. ONE OF THE QUESTIONS I HAVE IS WHETHER OR NOT WE COULD GET STARTED, ON THE SAME PAGE, SHARE THE BEST PRACTICES, NOT JUST HERE OR WITHIN THE NIH BUT KIND OF ACROSS THE TEAMS, GROUPS ORGANIZATIONS AND INSTITUTIONS THAT ARE CURRENTLY WORKING ON THIS TO GET A PLATFORM, AT LEAST A COMMON LEXICON, YOU KNOW, A LANGUAGE, A CHART, A SOMETHING THAT GETS US ALL STARTED. SO -- AND THIS IS GOING TO SOUND VERY SELF -- >> THIS IS GOING TO SOUND VERY SELF SERVING AND IT IS, BUT WE HAVE FELT LIKE, BECAUSE WE REPRESENT HUNDREDS OF MILLIONS OF PATIENTS, AND THAT'S WHO WE HAVE IN OUR MEMBERSHIP AGO WITH ALALONGWITH ALL THE OTHER STAKEHOLDER, WE ACTUALLY DEVELOPED A PROPOSAL TO DO EXACTLY THAT, TO WORK WITH ALL THE STAKEHOLDERS, FOR ONCE AND FOR ALL TO DEFINE PATIENT ENGAGEMENT FOR DIFFERENT PURPOSE, WHETHER IT'S FDA OR PCORI OR I KNOW DUKE UNIVERSITY, CTTI, THEY'RE LOOKING AT THIS, AND REALLY HAVE EVERYBODY TOGETHER, WE HAVE A WHOLE PROFESSIONAL WAY WE DO A DIALOGUE EVENT, WE DO AN ANALYSES, AND WE DO A LITERATURE REVIEW OF WHAT SEEMS TO BE BEST PRACTICES. SO I JUST WANT TO SAY, SOMEBODY HAS WRIT E WRITTEN IT DOWN ABOUT HOW IT ALL MIGHT BE DONE AND I WILL AT LEAST SHARE IT. >> LET ME ADD ON THAT, WE CAN DID HAVE, IN ONE OF THE BRAINSTORMING DISCUSSIONS, A CONVERSATION ABOUT IF NCATS IS ALREADY HAVING MEETINGS WITH SAKE HOLDERS AND W WE KNOW THAT IT HAPPENS THROUGH THE RARE DISEASE WORK AND CERTAINLY THE CTSA FOLKS GET TOGETHER, CAN YOU REPURPOSE OR SHARE ACROSS THESE BORDERS A MEETING OR TWO A YEAR WHERE YOU'RE BRINGING IN DIFRENT PATIENTS AND DISEASE FOUNDATIONS, SO IT'S KIND OF LEVERAGING OFF OF WHA YOU JUST TALKED ABOUT, FREDA, AND LISTEN TO THEM, NOT TALK AT THEM. SO CAN YOU STRUCTURE IT ACROSS A DAY OR TWO OF DIFFERENT OPPORTUNITIES TO INFUSE THE PATIENT PERSPECTIVE. AND USE THAT AS A MARKER FOR NCATS ON A YEARLY BASIS TO KIND OF DELINEATE WHAT YOUR PROGRESS IS. BUT PLEASE DON'T PUT A BUNCH OF PEOPLE UP AT THE PODIUM AND HAVE THEM TALK, TALK, TALK FOR 16 HOURS BECAUSE THAT'S NOT WHAT PEOPLE NEED. THEY NEED A FORUM TO ACTUALLY COME AND TELL YOU WHAT SHOULD HAPPEN, THEN YOU GUYS CAN GO OFF WITH YOUR TEAM LATER AND DECIDE WHAT YOU'RE GOING TO DO WITH IT. BUT I THINK IT'S WASTE OF THEIR TIME TO COME AND GET LECTURED TO. I'M NOT JUST PICKIN ON YOU, BUT I THINK IT'S TEMPTING TO DO THAT. WELL, GREAT, WE'RE GOING TO SHOW EVERYBODY WHAT WE'RE DOING. NO, NO, THAT'S FINE, DO THAT QUICKLY, BUT -- >> THAT'S ACTUALLY WHY, I THINK, FOR US THIS IS SO DIFFICT BECAUSE I HAVEN'T WANTED TO DO THAT. THAT WOULD HAVE BEEN EASY. IF THAT'S WHAT PATIENT ENGAGEMENT MEANS, WE WOULDN'T NEED A SUBCOMMITTEE. WE'D JUST TRUCK THEM IN, TALK TO THEM, TRUCK HEMIOUT AGAIN. BUT THAT'S WHAT WE DON'T WANT TO DO. SO THEN THE QUESTION IS -- I HAD THIS THOUGHT THAT WE HAVE TO, AS PART OF THE WORK YOU ALL DO, WE HAVE TO REPORT EVERY YEARS OF INVOLVEMENT OF WOMEN OF MINORITIES IN OUR CLINICAL TRIALS, SO IT'S SOMETHING WE T THIS. IT WOULD BE INTERESTING TO TRACK HOW WE'RE DOING IN THIS AREA. ONE IS STATUTORY, THE OTHER ONE ISN'T, BUT IT'S AN INTERESTING THING IF WE ESTABLISH THIS AS A -- WE HAVE TO KIND OF AGAIN DO WHAT WE DID WITH THE CTSA, IS WHAT WE'RE TALKING ABOUT, TO ESTABLISH A T 0. AND THEN HAVE MEASURABLE OBJECTIVES FOR WHERE WE'RE GOING. >> SO THE OTHER THING IS, I KNOW WHEN NCATS WAS BEING TALKED ABOUT IN THE EARLY DAYS AND CONSIDERED, ONE OF THE IDEAS WAS WHAT COULD NCATS OFFER UP TO THE OTHER INSTITUTES, RIGHT? SO I THINK THERE'S A CONVENING ABILITY THAT IT COULD GIVE NCATS IN TERMS OF AS MYRL SAID, THERE'S A MULTITUDE OF THINGS GOING ON ACROSS THE INSTITUTES AND YOU DON'T WANT TO BOYLE THE BOIL THE OCEAN. YOU COULD PREDICT A LOT BUT THE INSTITUTES AREN'T ABLE TO CONVENE WITH EACH OTHER THAT MUCH ON THIS PARTICULAR TOPIC, SO PUT A STAKE IN THE GROUND AND SAY, ALL RIGHT, WE'RE GOING TO DO IT, AND START SOME INTRAMURAL CONVERSATION ABOUT THIS. BECAUSE YOU KNOW, TO FREDA'S POINT, CAN WE PUT IT TO BED ONCE AND FOR ALL? I GUESS I'M NOT OF THE MINDSET THAT WE'RE GOING TO BE ABLE TO DO THAT, AND I THINK CHARACTERIZING IT IS FINE, BUT I THINK PART OF THE -- MAYBE A LITTLE BIT OF FRUSTRATION, SOME OF OUR COMMITTEE MEMBERS FELT IS THAT THEY'RE ALREADY DOING IT, THEY'RE OFF TO THE RACES. MY GOSH, THEY'VE DRUGS HA HAVE BEEN APPROVED. THEY'RE ON TO THE NEXT SET OF CHALLENGES WHICH IS HOW ARE THEY GOING TO PAY FOR THEM ALL, IS THE SYSTEM GOING TO ABSORB THEM, WHAT ABOUT THE RE LA FRI PARADIGM? SO SOMETIMES I THINK STOPPING AND CHARACTERIZING WHAT IS THIS FEELS LIKE A STEP BACK FOR SOME AND YOU'RE GOING TO HAVE TO ACCOMMODATE IT ALL OUT THERE. SO -- >> MARGARET, I WOULD SAY IT'S CHARACTERIZING IT WITH MULTIPLE CHARACTERIZATIONS IN THE SENSE AT A VERY HIGH LEVEL, WHAT IS IT WE THINK IT MIGHT MEAN, BUT THEN HOW IT BECOMES IMPLEMENTED WITHIN ALL OF THOSE DIFFERENT ENTITIES IS DIFFERENT. THAT'S WHY I SAID, YOU IDENTIFY THE TASK, WHAT YOU'RE GOING TO MEASURE, WHAT YOU WANT TO ACCOMPLISH, AND THEN YOU FIND THE RIGHT PEOPLE AND TRAIN THEM TO THOSE CORE CAN COMPETENCIES. SO IT LOOKS DIFFERENT BUT THE WAY WE THINK ABOUT IT MIGHT BE A LITTLE MORE FOCUSED. >> WE WENT BIG, THEN WE WANT BACK SMALL AGAIN, AND I THINK WE'LL PROBABLY CONTINUE TO DO THAT. I THINK WE JUST GOT A LITTLE OVERWHELMED WITH TRYING TO UNDERSTAND THE LANDSCAPE WHICH IS SO ENORMOUS, EVEN WITHIN THE WALLS OF THE NIH, LET ALONE EVERYBODY ELSE OUT THERE WHO'S TALKING AND DOING THINGS ON IT. BUT YOU HAD YOUR FINGER ON THE RIGHT PULSE, THAT'S FOR SURE. >> FIGURE ON THE AORTA, PERHAPS. OKAY. SO THANK YOU, THERE'S A LOT MORE TO SAY ABOUT THIS, BUT IT'S ONE OF MY FAVORITE TOPICS, WE COULD GO ON AND ON FOR A WHILE, BUT I WILL RESIST THAT TEMPTATION AND GO ON TO THE PARTNERSHIPS WITH PHARMACEUTICAL AND BIOTECHNOLOGY COMPANIES AND VENTURE CAPITAL FIRMS. FREDA TELLS ME ANKIT IS GOING TO START, HE MAY SAY FREDA IS GOING TO START. >> WHO'S ON FIRST? >> TEAM SCIENCE. ALL RIGHT. >> SO THANKS CHRIS. THANKS, FREDA. SO ON BEHALF OF FREDA, WE'LL TAKE YOU THROUGH QUARTERS' WORTH OF DISCUSSION OUR GROUP HAD, AND THANKS FOR COMMENTS HERE AS WELL, AS WELL AS THE SUPPORT FROM NCATS LEADERSHIP AND NSTAT, DAN, WILLIE PORTILA FOR THEIR EFFORTS AS WELL. SO I THINK THE LAST TIME WE HAD A BRIEF REPORT, WE WERE REALLY KIND OF WRAPPING OUR ARMS AROUND WHAT THE MISSION WAS FOR THE PHARMA AND BIOTECH COMMUNITY. IF I SAY IT IN ONE SENTENCE, FREDA MAY HAVE ANOTHER TO CHIME IN ON, IT'S REALLY HOW DO WE TAKE ALL OF THE GREAT ACTIVITIES OUT THERE AND LINK THEM UP WITH NCATS AND MAKE SURE THAT NCATS IS REALLY DOING WHAT IT DOES BEST AND GETTING THE MOST FOR THE RESOURCES THEY CAN PUT INTO PLAY. SO WE STARTED TO BRAINSTORM A LITTLE BIT, BUT I THINK QUICKLY BECAME APPARENT THAT TO THE POINT THAT MYRL AND PEGGY MADE WAS THAT THE UNIVERSE THERE IS SO BROAD THAT WE WANTED TO GET A LITTLE DEEPER UNDERSTANDING. SO WHAT WE SET OUT TO CAN DO WAS REALLY WITH THE HELP OF NORA IN PARTICULAR, WAS TO REALLY SURVEY THE NIH AND RELATE ORGANIZATIONS TO SEE WHAT INTERESTING COLLABORATIONS THERE ARE WITH PHARMA, BIOTECH AND -- ALREADY, AND GROUPS WHERE WE COULD LEVERAGE WHAT'S ALREADY GOING ON. SO THE WAY WE THOUGHT ABOUT MAKING AN IMPACT HERE IS SOME FAMILIAR THEMES. ONE IS LET'S FIND SOME SMALL VICTORIES, QUICK WINS HA WE CAN TAKE SO THAT YOU CAN FACILITATE PHARMA AND BIOTECH INTERACTIONS. THE SECOND IS ARE THERE TO THE POINTS GIVEN, WE HAVE X AMOUNT OF RESOURCES AND MAYBE 10X THE NUMBER OF PROBLE TO SOLVE, SO ARE THERE PROGRAMS ALREADY EXISTING WHERE WE CAN LINK INTO AND FACILITATE. AND THEN THIRDLY, I THINK THAT LEAVES ROOM ON THE PLATE FOR ADDITIONAL APPLICATIONS. SO WHERE WE ARE TODAY, I THENG WE'VE I'THINKWE'VE IDENTIFIED SOME QUICK W INS AND HAD THOUGHTS OF THE CLAB COLLABORATIVE PIECE OF IT, AND WHAT WE'LL COME BACK TO YOU NEXT TIME WITH IS A SET OF ACTIONS TO REALLY PUT THESE INTO PRACTI AND WE'LL GIVE MORE OF AN UPDATE THEN. SO THAT'S KIND OF A 10,000-FOOT VIEW. MAYBE MOVING ON TO THE NEXT SLIDE, FREDA, BEFORE I MOVE ON, ANYTHING ELSE THAT YOU'D ADD TO THAT? OKAY. SO WHAT WE DID WAS WAS REALLY A SCAN OF TRANSLATIONAL PROGRAMS AT THE NIH AS I MENTIONED. THERE'S AN EXQUISITE AMOUNT OF DETAIL THAT HEAF PUT TOGETHER THAT I'M HAPPY TO SHARE WITH THE GROUP. I THINK THE UPSHOT OF IT REALLY IS THERE ARE A VARIETY OF PROGRAM WITHIN OTHER NIH INSTITUTES THAT SEEK TO DO SIMILAR THINGS IN TERMS OF ADVANCED TRANSLATION AND WORK WITH PHARMA AND BIOTECH, IN FACT I WAS HEARING OVER LUNCH OF INTERESTING COLLABORATIONS WITH CTSAs DIRECTLY. SO THERE'S A LOT THAT WE CAN DO. I HINGE IN OU THINK IN OUR OPINION, THE NCATS MISSION IS TO REALLY LINK UP THERE. I THINK THE PREVIOUS PRESENTATION TALKED ABOUT NCATS AS SORT OF A CONVENING AUTHORITY, BEING SORT OF DIFFERENT AND SORT OF REALLY POE CUSSE ON THIS VERY TOPIC TO BRING GROUPS TOGETHER. SO FOR US, WE SAID LET'S FIND THOSE INTERESTING GROUPS, NOW WE'VE IDENTIFIED THEM. THE SECOND STEP IS GOING TO BE, HOW DO WE BEST ENGAGE THEM. I'LL KEEP THIS SHORT BECAUSE I'D LOVE TO GET OPINIONS FROM REST OF THE GROUP ABOUT HOW WE BEST DO THAT. BEFORE I GO THERE, FLIPPING ON TO THE NEXT SLIDE, IN THE QUICK WINS DEPARTMENT, WHAT WAS REALLY EVIDENT CERTAINLY FROM MY REAL LIFE EXPERIENCE AND FREDA'S IS THAT THERE ARE A NUMBER OF INSTITUTIONS IN THE FOR-PROFIT DRUG DEVELOPMENT SECTOR THAT WANT TO WORK WITH NIH IN PARTICULAR, AND THIS IS NOT A PARTICULAR THAT'S ENDEMIC TO NIH, IT'S ALSO ACADEMIC INSTITUTIONS IN GENERAL. THERE IS A LONG HARD PROCESS TO TRY TO FIND WAYS TO WORK TOGETHER. I THINK THE OTHER EXAMPLE WAS RY STREAMLINING THAT PROCESS IN MORE OF AN AD HOC WAY. I THINK THERE ARE, ESPECIALLY WITH NCATS FOCUSED ON THIS VERY MISSION, QUICK WAYS THAT ONE COULD TRULY THINK ABOUT DEVELOPING A PROCESS THAT'S A SORT OF MODIFICATION OF EXISTING NIH PROCESSES, SO ONE COULD AT LEAST WORK WITH NCATS A LITTLE MORE -- IN A LITTLE MORE FLUID WAY THAN SOME OF THE OTHER INSTITUTES THAT WE WORK WITH. SO FROM A STRATEGIC PERSPECTIVE, THERE'S A LOT OF IMPACT ONE COULD MINE FROM DOING THAT. I THINK THE NEXT REPORT, WE'LL BE REALLY THINKING ABOUT HOW WE DECIDED TO DO THAT. SO WHERE DID WE END UP WITH THAT? THERE ARE A FEW VERY GOOD NEAR TERM EXAMPLES. ON THE ONE SIDE, THERE ARE EXISTING PROGRAMS THAT WE SHOULD LERCHG IN TERMLEVERAGE IN TERMS OF TRYING TO GET OUR COMMON MISSION TOGETHER. AT THE SAME TIME, THERE'S OTHER GROUPS DOING THE SAME THING. NCATS HAS BUILT A VERY NICE SET OF -- AROUND RARE DISEASES, EVEN THE PATIENT E ENGAGEMENT FACTOR. FINDING WAYS TO WORK BETTER WITH THESE GROUPS HA WANT TO DO THESE THINGS, CHRIS IS SORT OF -- THE PICTURE THAT SAYS A THOUSAND WORDS, EXACTLY. MESHING TOGETHER IS REALLY WHAT WE LIKE TO DO. SO THAT'S THE WHAT IN OUR OPINION? I THINK THE HOW COMES NEXT, AND THAT'S REALLY WHERE WE ARE IN TERMS OF TRYING TO THINK ABOUT THE SUBCOMMITTEES. SO IF I KIND OF SUMMARIZE IN TERMS OF THERE'S SOME QUICK PROCESS WINS THAT WE CAN MAKE AS A GROUP, SECONDLY, THERE ARE A NUMBER OF GROUPS AND PROGRAMS THAT WE CAN TAP INTO ALREADY. I THINK GIVEN THE RESOURCE CONSTRAINTS WE HAVE, THAT'S PROBABLY THE SMARTEST WAY TO THINK ABOUT PHARMA BIOTECH VC INTEGRATION. THERE ARE RESOURCES OUT THERE TRYING TO DO SIMILAR THINGS AND GIVEN THAT WE ALREADY HAVE SORT OF A -- TO USE A COMMERL WORD -- FRANCHISE AROUND SOME OF THESE OPPORTUNITIES, CAN WE COMBINE RESOURCES AROUND THE SAME MISSION. AND THERE'S OBVIOUSLY A LOT OF INSTITUTIONAL WORK THAT NEEDS TO BE DONE TO MAKE THAT HAPPEN, BUT THERE'S ALREADY SOME GOOD EXAMPLES. SO I'LL STOP THERE, BUT MAYBE FREDA YOU HAVE OTHER THINGS YOU WANT TO ADD. >> I JUST WANTED TO ADD TOO, THAT WAS A FABULOUS SUMMARY OF KIND OF A LOT OF WORK IN DISCUSSION THAT WE'VE HAD. SO AS YOU ALL THINK ABOUT IT, BECAUSE WE INTEND TO TAP YOU MOVING FORWARD, ONE OF THE EXAMPLES THAT WE HAVE HERE IS AN IDEA, THE NTU PROGRAM. IF YOU THINK ABOUT IT, THERE ARE THREE POINTS OF INTERSECTION ON THIS. GETTING MORE COMPOUNDS IN RIGHT NOW, MOST OF OUR COMPOUNDS ARE COMING FROM LARGE AND MID SIZED PHARMA COMPANIES. WHAT IF WE COULD EXPAND THE BACK END AND GET MORE? RIGHT NOW ALL OF THE MONEY IS NIH FUNDING. IS THERE AN OPPORTUNITY FOR US TO COFUND SOME OF THESE EXPERIMENTS AGAINST THE COMPOUNDS, AND THEN THE THIRD LEG IS, WE HAVE A FAIRLY RESTRICTED ALTHOUGH BROAD SET OF INVESTIGATORS. IS THERE A WAY TO KIND OF AMPLIFY THE NUMBER OF INVESTIGATORS THAT HAVE ACCESS TO THEM, SO YOU COULD TAKE A SINGLE PROGRAM LIKE THIS, AND KIND OF PUSH IT OUT IN A NUMBER OF WAYS WITH COLLABORATION, AND AT THE PINNACLE WOULD BE COFUNDING TO EXPAND THE AMOUNT OF MONEY TO HAVE TO ADVANCE THESE MOLECULES. SO WE HAD SOME EXCITING DISCUSSION AROUND THAT, AND THEN THE SECOND THING IS, THERE ARE ORGANIZATIONS WHO HAVE COFUNDING MANDATES THAT ARE CHOMPING AT THE BIT TO DO SOME WORK WITH US, FINDING ON RAMPS FOR US TO DO THAT WORK IS REALLY GOING TO BE A PART OF WHAT WE NEED TO GET YOUR HELP ON IN THE NEAR TERM. DID I JUST KILL ALL CONVERSATION? >> I HAVE A QUICK QUESTION BECAUSE ONE OF THOSE OPPORTUNITIES UNDERWAY WAS ALPHA 1. AND AT SOME POINT, IT DOESN'T HAVE TO BE NOW, BUT JOHN WALL SH, CEO, IS ON OUR BOARD OF DIRECTORS, BUT I'VE KNOWN FOR A WHILE THAT HEAR FUNDING OR CO-FUNDING A PERSON, AN INDIVIDUAL WHO'S NOW WORKING HERE, AROUND THOSE AREAS AND NONE OF US KNOW HOW THAT HAPPENED. HOW DID THAT OPPORTUNITY -- DID HE JUST COME AND SAY I WANT TO DO THIS OR WHAT? I DON'T HAVE TO KNOW, PU HOWEVER IT HAPPENED AND IF IT'S WORKING WELL, THAT'S, I THINK, WHAT FREDA IS TALKING ABOUT. GIVE THE PATIENT GROUPS AND OTHERS AN OPPORTUNITY TO CO-FUND, DOESN'T HAVE TO BE ABOUT THEIR DISEASE BECAUS WOULD BE THOSE PATIENT GROUPS THAT REALLY UNDERSTAND MULTIPLE CHRONIC CONDITIONS, EVERYTHING IS CONNECTED AND THEY WOULDN'T COME IN EXPECTING IT WOULD BE JUST ABOUT THEIR DISEASE. >> THE SHORT ANSWER IS YES, IT HAPPENED EXACTLY AS YOU'RE SAYING. THAT I KNOW JOHN AND HANNA'S HOPE MOM FOUND US ON THE WEBSITE OR I MET HER AT A MEETING OR SOMETHING. AND SO THEY FUND, BOTH OF THEM FUND FELLOWS AT NCATS THROUGH THE CONDITIONAL GIFT FUND. THAT'S HOW THIS WORKS. SO THEY ARE -- THEY'RE NAMED FELLOWS, HANNA'S HOPE FELLOW, ALPHA 1 FELLOW, WE'VE HAD MYOTO MYOTONIC DYSTROPHY FELLOW, ET CETERA, ET CETERA. THEY GET ACCESS -- YOU PROBABLY HAVEN'T SEEN THE LAB, BUT -- WE SHOULD HAVE A MEETING UP AT THE LAB SOMETIME BECAUSE I THINK YOU WOULD ALL ENJOY THAT, WE HAVE TO HAVE THAT, MAYBE FOR THE NEXT ONE, AND YOU DON'T HAVE TO DEAL WITH SECURITY EITHER. THERE'S FREE PARKING AND, ET CETERA, ET CETERA. BUT ANYWAY, THIS HAPPENED VIA HAPPENSTANTIAL INTERACTIONS WITH VARIOUS DISEASE GROUPS, SO THE QUESTION IS, SHOULD WE -- THIS IS REALLY A BIOTECH PHARMA VC THING BUT IT'S ALSO A PATIENT ENGAGEMENT THING, SHOULD WE GENERALIZE THIS IN SOME WAY? IT CLEARLY WORKS. THE EXAMPLES I COULD GIVE YOU WOULD REALLY BLOW YOUR MIND AS FAR AS HOW -- WHAT'S BEEN ACCOMPLISHED THROUGH THESE, BECAUSE WHAT HAPPENS IS THAT THEY GET ACCESS TO THE ENORMOUS CAPITAL INVESTMENT AND EXPERTISE UP THERE WITH A SMALL MARGINAL INVESTMENT ON THEIR PART, AND NCATS HAS ACCESS NOT ONLY TO THE SMALL AMOUNT -- RELATIVELY SMALL AMOUNT OF MONEY THAT PAYS FOR A FELLOW, BUT THAT GETS US ACCESS TO THEIR ENTIRE SCIENTIFIC ADVISORY BOARD, AND THAT'S WHY IT REALLY WORKS, BECAUSE IT'S THE SCIENTIFIC ADVISORY BOARD THAT YOU MATCH UP WITH THIS ENORMOUS TRANSLATIONAL EXPERTISE AND THINGS HAPPEN VERY QUICKLY IN THAT CASE. >> I WOULD SO SUPPORT HAVING THAT OPPORTUNITY CODIFIED AND MADE KNOWN. I MEAN, YOU KNOW, THOUGHTFULLY SO THAT PEOPLE DON'T HAVE AN EXPECTATION, BUT YOU HAVE EXAMPLES HERE WHERE PEOPLE FOUND OUT ABOUT IT. THEY HAPPENSTANCE -- IF IT'S WORKING, WHY WOULDN'T THAT BE ONE OF THE MOST PROMINENT RIGHT NOW THINGS THAT YOU WANT TO SAY WE'RE DOING THIS RIGHT, AND I MEAN, WE'RE LINKED TO AT LEAST 50 OF THOSE NATIONAL GROUPS. WE HAVE ONE PERSON SAYING, WELL, I DID THIS, AND THEY'RE ALL GOING, WELL, HOW? YOU KNOW? SO I WOULD REALLY ENCOURAGE YOU TO PUT IT TOGETHER. >> ONE OF THE PROBLEMS YOU MENTIONED WAS THE ABSENCE OF A MECHANISM FOR SPINNING OUT OR COMMERCIALIZING AN OPPORTUNITY, BUT THIS IS ONE AREA WHERE PATIENTS OR GROUPS HAVE STARTED MAKING SOME INTERESTING ADVANCES. RECENTLY WE TALKED TO THE JUVENILE DIABETES FOUNDATION AND THEY'RE HOOKED UP WITH A VC, AND THEY HAVE A MECHANISM WHERE THEY TAKE NIH MONEY, JDRF MONEY, AND IF IT'S PROMISING, THEN THE VENTURE CAPITAL MONEY COMES IN AND THEY'RE REALLY TRYING TO ENLIST THE PHARMA COMPANIES AS PART OF THIS FUND. >> YEP. >> SO I ACTUALLY THINK THIS IS AN AREA FOR A REALLY FRUIT PULL ANFRUITFULAND CREATIVE DEAL MAKING, LEVERAGING FEDERAL FUNDING, FUNDING FROM THE FOUNDATIONS, AND USING VC MECHANISMS TO LINK TO THE PHARMA INDUSTRY. >> [INAUDIBLE] >> SO MOST OF IT BY DESIGN IS MORE TRANSLATIONAL, SO A GREAT EXAMPLE, JUST FOR SAKE OF EXAMPLE THERE, HAVING SPENT SOME TIME LOOKING AT RARE DISEASE INVESTMENTS, WE'VE SEEN MOST OF THE TREND PROGRAMS. WHAT WOULD BE GREAT IS IF WE WERE BETTER INTEGR INTO THAT PROCESS OF DEVELOPMENT. WE'VE SEEN THESE COMPANY, THEY DO THESE PARTNERSHIPS, THEY DO GREAT WORK BUT THEY'RE KIND OF OFF LIMITS. IF THERE'S A WAY TO GET BETTER INTEGRATED, AT SOME POINT THEY'RE GOING TO HAVE TO RAISE ADDITIONAL CAP AL TO GET CLOSER TO THE CLINIC. I THINK THAT'SA VERY INTRIGUING WAY TO DO IT. OTHER GROUPS IS DONE IT MORE IN A BROADER FASHION, THEY HAVE FUND AND ALL THEY DO IS INVEST IN THE PARTICULAR INSTITUTION'S WORK, SMALL OR LARGE BITS, AND THERE ARE GROUPS OUT THERE, AND WE'VE SPOKEN TO SOME OF THEM. THAT'S NOT A BAD WAY FOR US TO LEVERAGE THE IMPACT WE HAVE, BECAUSE AGAIN, WE HAVE A FINITE AMOUNT OF RESOURCES AND DO EVERYTHING WITHIN OUR OWN WALS. OWN WALLS. >> JUST TO PERHAPS KIND OF ADD TO THAT, SO WITH NCATS, THE LEUKEMIA LYMPHOMA SOCIETY AND KU, WE HAVE THIS PARTNERSHIP CALLED THE LEARNING COLLABORATIVE WHICH IS FOCUSED ON DISCOVERING/DEVELOPING NEW TREATMENTS FOR BLOOD CANCERS, >> Dr. Anderson: IN THAT PARTNERSHIP MODEL, LEUKEMIA LYMPHOMA SOCIETY IS TAKING ON THE ROLE OF FINDING THE INDUSTRY PARTNER, AND AS YOU MENTIONED, SO LITERALLY I THINK FOR ONE PARTICULAR PROJECT TOGETHER, I THINK WE'VE RAISED ABOUT 2.6 MILLION, FOR ONE PARTICULAR PROJECT. WELL, AND WE'VE WRITTEN THIS PARTNERSHIP UP AND WE'RE LITERALLY NOW IN THE PROCESS OF WRITING UP OUR LEARNINGS FOR THE FIRST THREE YEARS THAT WE HOPE TO PUBLISH. WELL, WHAT WE'VE DONE BETWEEN NCATS AND KU AND CHILDREN'S MERCY HOSPITAL IN KANSAS CITY IS WE WANTED TO TEST THAT COLLABORATION MODEL BY CREATING A COLLABORATIVE AROUND SARCOMAS. AND THE REASON -- ONE OF THE REASONS WE CHOSE THE SARCOMAS IS THERE IS NO LEUKEMIA LYMPHOMA SESOCIETY, NO VERSION OF THE LEUKEMIA LYMPHOMA SOCIETY WITHIN THE FACE OF SARCOMAS. SO WE'VE INTENTIONALLY TAKEN THAT ON. AND WHAT WE'RE THINKING WITHIN THE COLLABORATION IS, IF WE HAVE A DRUG, A NEW DRUG TREATMENT THAT'S POTENTIALLY PARTNERRABLE, WHAT WE'RE GOING TO DO IS GO OUT TO FIND A BIOTECH EXPERT THAT WE CAN BRING ON TEMPORARILY FOR THAT PROJECT AND HELP US PARTNER THAT WITH THE RIGHT PARTNER. SO I JUST WANTED TO SHARE THAT. >> INTERESTING CONVERSATION. >> THAT'S A BRILLIANT EXAMPLE OF HOW YOU NEED TO THINK ABOUT THE WORLD WHERE THERE MIGHT NOT BE A DISEASE FOUNDATION OR A ROBUST PATIENT ORGANIZATION ON THE DISEASE THAT YOU UNFORTUNATELY GET. AND I DON'T THINK WE SHOULD EXPECT EVERYBODY TO HAVE TO RUN OUT AND CREATE THEIR OWN, EVEN THOUGH MANY UNFORTUNATELY HAVE TO DO THAT. SO I LOVE THAT YOU'RE GOING TO TAKE THE LEARNING FROM THIS AND I THINK THAT'S A ROLE THAT NCATS CAN DO, IS TRY TO FIGURE OUT HOW DO WE SPREAD THIS WEB SO THAT WE'RE TAKING THE BEST PRACTICES THAT ALL OF THIS REPRESENTS, AND GIVING IT TO THE ENTIRE SYSTEM. BECAUSE THAT'S WHAT WE ALL ARE HOPING FOR. >> ANOTHER FANTASTIC CONVERSATION THAT WE NEED MORE TIME FOR, BUT I WANT TO HAVE ENOUGH TIME FOR MED TECH, SO PAUL, DO YOU WANT TO TAKE THAT ON? >> WE'LL DO A MINI PRESENTATION THAT CONSISTS OF TWO MICRO PRESENTATIONS. FRANK DOUGLAS CO-CHAIR IS ON THE PHONE. FRANK, ARE YOU OKAY IF I GO FIRST HERE? >> YES, GO AHEAD, PLEASE, PAUL. >> GREAT. SO OTHER COMMITTEE MEMBERS, HERE MARGARET AND SCOTT ARE ON THIS COMMITTEE, BOB TEPPER AND SUSIE GEL, WHSUESIEGEL WHO AREN'T HERE TODAY. WE HAVE SOME GREAT EXTERNAL REPRESENTATION. FDA, CMS, THEN BILL IS HERE, BACK IN THE BLUE COAT IN THE BACK. >> [INAUDIBLE] BILL IS AT THE NATIONAL INSTITUTE FOR BIOMEDICAL IMAGING AND BIOENGINEERING. DID I GET THAT RIGHT? > SO JUST TO REFRESH EVERYBODY'S RECOLLECTION, THIS COMMITTEE WAS FOUNDED OUT OF CHRIS' CONCEPT THAT THERE WAS A SKITOMA OR BLIND SPOT IN NCATS, JUST BY VIRTUE OF ITS HISTORY AND CULTURE IN THE WAY IT GREW UP WITH RESPECT TO MED TECH. BY MED TECH, WE MEAN MEDICAL DEVICES AND DIAGNOSTICS. I'D SAY EVEN THE CONVERSATION WE JUST HAD, YOU KNOW, WE WEREN'T REALLY THINKING ABOUT MEDICAL TECHNOLOGY COMPANIES, WE DIDN'T T ABOUT THAT. SO OUR GROUP DECIDED THAT THE FIRST THING TO DO TO GET A LITTLE MORE ACCURATE IDEA OF WHAT ACTUALLY IS GOING ON NOT œIS TO DO A LANDSCAPING EXERCISE ABOUT WHAT PERCENTAGE, WHAT KIND OF FUNDING IS NIH DEVOTING OH TO TO TRANSLATIONAL WORK IN MEDICAL DEVICES/DIAGNOSTICS COMPARED TO BIOTECH, BIOPHARMA-RELATED PROJECTS. SO WE ARE DEEPLY INTO THAT. BILL HAS BEEN TREMENDOUSLY USEFUL, AS HAS THE NCATS STAFF, SO DAN HAS MOBILIZED A COUPLE OF FOLKS, ANTON DOWN AT THE END, AND THEN RALPH BASHNER ALSO. WHERE WE'RE AT IS WE'VE DONE A REASONABLY DEEP DIVE ON THE MED TECH SIDE OF THINGS, COURTESY OF BILL AND IBIB, AND YOU MAY BE INTERESTED TO KNOW THAT THERE ARE 2,364 PROPOSALS OF VARIOUS SORTS RELATED TO MED TECH THAT ARE FUNDED ACROSS NIH. THAT'S AN APPROXIMATE NUMBER RIGHT MOW. WE'RE DOING DILIGENCE ON BUFFING THAT UP. WE ARE WORKING ON THE -- IN THE NEXT FEW WEEKS, WE'LL HAVE AN ACCURATE NUMBER RELATED TO BIOPHARMA BIOTECH. WE'RE GOING TO PARSE THAT ACCORDING TO INSTITUTE, SUBDIVISIONS OF THOSE TECHNOLOGY SECTORS, TYPES OF GRANTS, LILY HAS HELPED US TO DO A PRELIMINARY DIVE INTO SBRS, TTR WILL LOOK AT THAT. SO THAT'S OUR FIRST ORDER OF BUSINESS, THEN WE WILL BE COMING BACK AFTER WE HAVE THAT ESTABLISHED TO START MAKING SOME SPECIFIC RECOMMENDATIONS. FRANK RECENTLY ATTENDED A MEETING HE'LL TELL YOU ABOUT, I THINK HE HAS A COUPLE OF -- YEAH, THE SLIDES ARE UP, FRANK, IN THE ROOM, SO WHY DON'T WE SEGUE TO YOU. >> OKAY. THIS AS YOU RECALL, THE BROOKINS INSTITUTION HAD A WORKSHOP ON DEVICES. PAUL DEDICATED THAT TASK TO ME, TO ATTEND THE WORKSHOP. IT WAS AN EXCELLENT WORKSHOP. DAN AND CHRIS WERE ALSO THERE. SO DAN AND CHRIS, YOU MAY WANT TO SUPPLEMENT THE COMMENTS THAT I MAKE. I'VE PUT TOGETHER A FEW SLIDES TO CAPTURE THE DISCUSSIONS, COMMENTS, FROM A THEMATIC PERSPECTIVE THAT OCCURRED IN THE WORKSHOP. AND I TRIED TO DO IT FROM THE LENS OF NCATS IN A ACCEPTS OF WHASENSE OF WHATWOULD ACCELERATE A REAL SE NSE, WOULD ACCELERATE TRANSLATION, AND ONE OF THE BEST WAYS TO ACCELERATE TRANSLATION IS, OFTEN, TO SEE THE SUCCESS AND DIFFERENT APPROACHES TO GET DRUGS, DIAGNOSTICS INTO THE SERVICE OF THE PATIENT. THERE WERE A NUMBER OF THEMES WHICH I'VE CAPTURED HERE. THE DISCUSSION, I WOULD PROBABLY DESCRIBE IN THE CONTEXT OF THE DISCUSSIONS YOU'VE BEEN HAVING MOST OF THE DAY AS NOT DISRUPTIVE, BUT IMPORTANT IN THAT THERE WAS A LOT OF CONSENSUS IN THE ROOM, AND IN THE ROOM, COMMENTS FROM FDA, CMS, NCATS, NIH, PARTICULARLY NCATS, VENTURE CAPITAL GROUPS WERE ALSO REPRESENTED IN THE ROOM. SO IF WE HAVE THE FIRST SLIDE UP, THERE IS A LOT OF DISCUSSION AROUND HOW TO IMPROVE THE REGULATORY REIMBURSEMENT PROCESS SO WE STOP LOSING FIRST IN HUMAN STUDIES TO EUROPE AND OTHER COUNTRIES. AND AS YOU KNOW, OFTEN IN THE DEVICES PARTICULARLY, THE -- IS ACHIEVED BEFORE WE GET THE REGULATORY APPROVAL IN THE U.S. SOME OF THE COMMENTS MADE AS YOU SEE IN THE SLIDES HERE WERE REALLY QUITE IMPORTANT. ONE OF THEM, FOR EXAMPLE, THIS DISCONNECT BETWEEN APPROVAL AND REIMBURSEMENT CRITERIA, AS IT TURNS OUT, THERE ARE DIFFERENT CRITERIA FOR APPROVAL AND REIMBURSEMENT, SO THE SITUATION OCCURS NOT INFREQUENTLY, IN WHICH A COMPANY GETS REGULATORY APPROVAL, AND THEN CMS SAYS REIMBU YOU HAVEN'T DONE THE RIGHT STUDIES, YOU HAVEN'T MADE THE RIGHT COMPARISON. YOU CAN IMAGINE HOW DIFFICULT AND DISTRESSING THAT IS FOR COMPANIES. WE DISCUSSED PARALLEL/REGULATORY REIMBURSEMENT REVIEW. THEY HAD LAUNCHED A PILOT AROUND END STAGE RENAL DISEASE. I WAS A LITTLE BIT DISAPPOINTED, I DID POSE THE QUESTION TO FIND OUT HOW THAT WAS GOING, AND THAT I WAS A LITTLE BIT DISAPPOINTED BECAUSE IF I RECALL HIS ANSWER CORRECTLY, WHAT HE BASICALLY SAID IS THAT THE COMPANIES WERE NOT AS ENTHUSIASTIC ABOUT IT AS HE HAD HOPED. THAT'S HOW I SAW THE BOTTOM LINE OF IT. THAT WAS WHAT WE DID IN THE EARLY DAYS OF BIOTECHNOLOGY WHEN CEBR WAS BORN, WE ACTUALLY HAD THE FDA LEARNING AT THE SAME TIME AS THE INNOVATOR WAS LEARNING THE DEVELOPMENT OF THESE NEW BIOTECHNOLOGY APPROACHES. SO THE QUESTION WAS, SHOULD THERE BE A GROUP LIKE HAD THAT, A REVIEW GROUP LIKE THAT IN THE FDA FOR INNOVATIVE DEVICES, SO THAT THEY COULD LEARN AT THE SAME PACE AS THE INNOVATOR AND ACCELERATE APPROVAL. A LOT OF DISCUSSION AROUND POST MARKETING SURVEILLANCE, AND I'LL COME BACK TO THAT ON A LATER SLIDE. ONE OF THE THINGS WHICH I ACTUALLY WAS NOT AWARE OF IS THAT INSURERS REIMBURSE DEVICES BASED ON HOW THOSE DEVICES ARE DESIGNATED OR CATEGORIZED BY IRBs. SO IF IRB DESCRIBES THE PARTICULAR DEVICE APPROACH AS EXPERIMENTAL, IT WOULDN'T GET REIMBURSEMENT. IF WE CAN GO TO THE NEXT SLIDE, ADVERSE EVENT TRACKING, AS I SAID, I'LL COME BACK TO THIS ISSUE. A LOT OF DISCUSSION AROUND POST MARKETING SURVEILLANCE AND ALSO SHOULD WE NOT DO SOMETHING WITH RESPECT TO CHANGING THE LENGTH OF TIME FOR THE PREMARKET APPROVAL PROCESS AND DO A SHORTER TIME FRAME, AND WE HAVE HAD THIS DISCUSSION ACTUALLY ON THE DRUG SIDE FOR MANY YEARS, AND COUPLE THAT WITH A LONGER TIME PERIOD OF POST MARKETING SURVEILLANCE. QUESTION OF THE NATIONAL REGISTRY OF -- >> FRANK, THIS IS PAUL. YOU HAVE THE DISADVANTAGE OF NOT BEING IN THE ROOM, BUT I JUST SAW A NOTE THAT WE NEED TO SLIDE IN TO VOTING PRETTY QUICKLY HERE, SO I'M SORRY TO SAY BUT WE PROBABLY SHOULD GET TO A SUMMARY. >> OKAY. THEN LET ME GIVE YOU -- I HAVE THREE MORE SLIDES AND YOU CAN READ THE SLIDES FASTER THAN I CAN SAY THEM. ON THAT SLIDE WE'RE LOOKING AT, I'LL CALL ATTENTION TO POINT B. WHICH I THINK IS SOMEWHAT DISRUPTIVE. COULD THERE BE AN ALTERNATE P FOR DEVICES FOR DISEASES, RARE DISEASES, AND PEDIATRICS. NEXT SLIDE, I THOUGHT THAT HFS A QUESTION AS A MATTER OF FACT THAT COULD LEAD TO DISRUPTIVE TYPE SOLUTINS. AND THAT IS THE QUESTION WAS ASKED, ARE WE USING A DRUG APPROVAL PARADIGM FOR DEVICES AND IS THAT APPROPRIATE. I WOULD NOTE ON THAT SLIDE TO BE COMPLETE THAT CDRH IS INDEED LOOKING AT THE FEASIBILITY OF ADJUSTING THE PREMARKET APPROVAL POST MARKET SURVEILLANCE BALANCE. AND THE FINAL SLIDE, A PROPOSAL, I MUST CONFESS THAT I SORT OF PUSHED IT AT MY TABLE SUBGROUP, IS THERE A POSSIBILITY OF USING A TYPE OF REPURPOSING APPROACH FOR OFF LABEL OR NEW USES OF DEVICES USING THE PARADIGM THAT PRESENTLY EXISTS FOR REPURPOSING. AND FINALLY, I'D JUST LIKE TO CALL YOUR ATTENTION TO -- THAT IS PRESENTLY OUT, THE RESEAR EVALUATION COMMERCIALIZATION HUB, AND IF YOU'RE NOT AWARE OF IT, YOU REALLY SHOULD LOOK AT THAT, IT REALLY FOCUSES ON FTTRs, UP WITH O ONE OF THE THINGS WE WERE VERY CONCERNED ABOUT, HOW WE COULD INCREASE THE SCOPE OF STTRs. UNFORTUNATELY THEY'RE ONLY PLANNING TO DO THREE HUB, BUT I'M ACTUALLY QUITE EXCITED BECAUSE INSTITUTE WIDE, THIS REACH, THIS RFV TELLS ME THAT A LOT OF THE THINGS THAT NCATS IS DOING IS BEGINNING TO GET SOME TRACTION. AND LET ME STOP THERE. DAN, CHRIS, IF THERE'S ANYTHING ELSE YOU'D LIKE TO ADD. >> I'D LOVE TO. UNFORTUNATELY WE'RE OUT OF TIME. I AM GOING TO HAVE AT THE NEXT -- I'M GOING TO TO HAVE LILY SEND AROUND SOME INFORMATION ABOUT REACH, BECAUSE SOMEBODY JUST ASKED ME ABOUT THIS THE OTHER DAY, IT'S A REALLY INTERESTING PROGRAM. SO THERE'S A LOT TO TALK ABOUT HERE, BUT WE REALLY -- WE'RE AT RISK OF LOSING FOLKS THAT WE NEED A QUORUM FOR FOR THE CLOSED SESSION, SO AT 4:30 AND WE'VE GOT A LOT TO DO BEFORE THEN. SO I'M GOING TO ASK PETRA TO VERY QUICKLY GO THROUGH A -- THANK YOU, FRANK. THANK YOU, PAUL. AND THANK YOU, THE REST OF THE SUBCOMMITTEE MEMBERS. PETRA HAS A PROPOSED -- A CONCEPT CLEARANCE FOR A PROPOSED CTSA INITIATIVES THAT I THINK WILL SOUND PRETTY APROPOS ACTUALLY. >> SO THIS IS FOLLOWING UP ON THE REPORT AND THE PURPOSE OF THIS IS TO OBTAIN CONCEPT CLEARANCE TO RELEASE FUNDING OPPORTUNITY ANNOUNCEMENTS TO IMPLEMENT SOME OF THESE TRANSITIONS AND TRANSFORMATIONS. SO THE GOAL HERE IS TO PROVIDE OPPORTUNITIES FOR UNIVERSITIES TO COMPETE FOR AWARDS UNDER THE CTSA PROGRAM. AND THIS IS CRITICAL AS YOU MAY KNOW BECAUSE THEY HAVE NOT BEEN FUNDING OPPORTUNITY ANNOUNCEMENTS SINCE 2012 BECAUSE NCATS WAS WAITING FOR THE REPORTS AND THAT GUIDANCE THAT CAME FROM THESE REPORTS. SO THE PLAN HERE IS TO IMPLEMENT GRADUAL CHANGE AS WAS DISCUSSED EARLIER, AND THE PROCESS IS THAT WE WILL CONSIDER THE RECOMMENDATIONS THAT WE JUST RECEIVED FROM THE WORKING GROUP TO GO THROUGH THEM CAREFULLY AND THEN MAKE THEM INTO STRATEGIC PLANS AND HAVE THEM BE INCORPORATED INTO THESE ANNOUNCEMENTS. WE ALSO OF COURSE TAKE INTO ACCOUNT AREAS OF INTEREST THAT HAVE BEEN IDENTIFIED BY THE CURRENT CTSA INVESTIGATORS AS PART OF THE STEERING COMMITTEE OR RATHER THE WORKING GROUPS. BASICALLY THE WAY WE LOOK AT THIS JUST BRIEFLY IS ALONG THE RECOMMENDATIONS IN THE IOM. AS YOU KNOW THE FIRST RECOMMENDATION IS TO STRENGTHEN NCATS LEADERSHIP AND AS YOU HAVE HEARD ALREADY, THERE HAS BEEN MUCH PROGRESS IN THIS ARENA THAT THE ESTABLISHMENT OF A SINGLE STEERING COMMITTEE, AND THEN THE STREAMLINING OF THE COMMITTEE STRUCTURE WHICH WAS ANOTHER KEY RECOMMENDATION. THE IOM REPORT ALSO WAS CALLING FOR BUILDING ON THE STRENGTH OF IUAL CTSAs ACROSS THE SPECTRUM, AND THE PLAN IS THERE TO ENCOURAGE FLEXIBILITY AND ALLOW EACH INSTITUTION TO BUILD ON THEIR UNIQUE STRENGTH AND INTERESTS, BUT ALSO TO STRENGTHEN NETWORK CAPACITY. SO THAT THE -- A BETTER POSITION FOR MULTISITE RESEARCH. THE FURTHER RECOMMENDATIONS WILL BE TAKEN INTO ACCOUNT SUCH AS THE IMPORTANT ONE THAT WAS MUCH DISCUSSED ALSO EARLIER TODAY, TO FORMALIZE AND STANDARDIZE THE EVALUATION PROCESS FOR THE WHOLE PROGRAM, TO ADVANCE INNOVATION IN EDUCATION AND TRAINING, TO ENSURE COMMUNITY ENGAGEMENT WHERE COMMUNITY IS NOT JUST GEOGRAPHICALLY DEFINED, AND ALSO TO STRENGTHEN RESEARCH RATHER THAN TO DIFFERENT STAGES IN LIFE. SO BASICALLY OUR NEXT STEPS WILL BE TO REFINE THE PLANS AFTER CAREFUL REVIEW OF THE WORKING GROUP REPORT AFTER LOOKING AGAIN AT WHAT THE INVESTIGATORS HAVE EXPRESSED AN INTEREST IN AND COME UP WITH, AND WE WILL THEN ISSUE AS POSSIBLE, REALLY, A SUITE OF FUNDING OPPORTUNITY ANNOUNCEMENTS, SO NOT JUST ONE. AND THESE WILL SOLICIT APPLICATIONS FOR SOMETHING THAT WILL BE MORE AKIN TO WHAT IS NOW REFERRED TO AS INSTITUTIONAL CTSA AWARDS BUT ALSO TRAINING AND SCHOLAR GRANTS AS WELL AS OPPORTUNITIES TO BUILD NETWORK CAPACITY. SO THIS IS ASKING FOR COUNCIL CONCEPT CLEARANCE. THANK YOU. ARE THERE ANY QUESTIONS? >> I HAVE A QUICK ONE. I KNOW WE'RE IN A HURRY, BUT I WAS INTERESTED THAT IT SAID IN THE FIRST SLIDE TO MAKE THIS AVAILABLE FOR UNIVERSITIES. IS THAT -- IS OUR LANGUAGE THAT NARROW OR COULD BE ORGANIZATIONS MAYBE? >> NO, ORGANIZATIONS. YEAH. CORRECT. >> OKAY. THANK YOU, PETRA. SO THE CONCEPT CLEARANCE IS ESSENTIALLY A PROCESS BY WHICH NCATS IS SEEKING PUBLIC ADVICE -- SO WHAT PETRA JUST DESCRIBED IN THE CONCEPT IS ESSENTIALLY THE BASIC PURPOSE, SCOPE AND OBJECTIVES OF THE CONCEPT OR THE PROPOSED INITIATIVE, SO WHAT WE WOULD LIKE COUNCIL TO DO IS TO CONCUR IN TERMS OF MOVING THIS INTO THE IMPLEMENTATION WHICH IS TO DEVELOP THIS INTO RFAs. UNLESS THERE ARE ANY QUESTIONS FOR DISCUSSION, THEN WE COULD ENTERTAIN A MOTION? NORA? SECOND? A IN FAVOR? GEORGE, FRANK, WE NEED -- >> I'M ONLINE. >> GEORGE? I HEARD A BEEP. >> FRANK ONLINE. >> HE'S STILL ONLINE? OKAY. DO WE HAVE A CONCUR FROM FRANK AND GEORGE? >> LET'S AFFIRM FRANK. I'M ONLINE. >> GREAT. ANYONE OPPOSED? ALL RIGHT. THANK YOU. >> OKAY. SO THANK YOU. WE ARE GOING TO ADJOURN THE OPEN SESSION, AND FOR THOSE THAT IT'S APPLICABLE TO, COME BACK FOR THE CLOSED SESSION. IF WE CAN DO THIS IN FIVE MINUTES, NOT AT 3:30, THAT WOULD BE GREAT, SO JUST TAKE A QUICK BIO BREAK. OR THOSE WHO AREN'T STAYING, THANK YOU VERY, VERY MUCH FOR BEING HERE, AND WE WILL BE IN TOUCH, LOOK FORWARD TO SEEING YOU NEXT TIME. AND FOR THOSE OF YOU WHO ARE STAYING FOR THE CLOSED SESSION, PLEASE BE READY TO GO IN ABOUT 5 MINUTES AND WE'LL START.