>> WELCOME EVERYONE. WELCOME TO COMMEMORATING 10 YEARS THROUGH SCIENCE AND THROUGH THE PANDEMIC WE'RE STRUGGLING WITH FEW OF US HAVE BEEN ABLE TO MEET AND ENGAGE LIKE WE HAVE IN THE PAST AND OUR DOORS ARE OPEN AND I'M EAGER TO WORK TOGETHER TO STRENGTHEN EXISTING RELATIONSHIPS AND FORGE NEW ONES THAT WILL CHANGE OUR TRANSLATIONAL SCIENCE WORLD AND I'M JONI RUTTER. PRIOR TO JOINING NCATS AS DEPUTY DIRECTOR I HAD THE PLEASURE OF SHAPING THE ALL OF US PROGRAM AND SERVED AS THE DIRECTOR OF SCIENTIFIC DIRECTOR AND NOW IT'S AN HONOR TO LEAD NCATS DURING PROBABLY THE MOST CRITICAL PUBLIC HEALTH CHALLENGE OF OUR TIME. WE'VE BEEN ALL IN CREATING AN ELECTRONIC HEALTH RECORDS ENCLAVE AND SUPPORTING CLINICAL TRIALS AS PART OF THE INVENTION AND VACCINES AND ACTIVE PROGRAMS AND DEVELOPING NEW ANTIVIRALS TO NAME A FEW THINGS WE'VE DONE. NCATS OPENED ITS DOORS IN 2011 TO MAKE TRANSLATIONAL MORE PREDICTIVE AND WE'RE NOW HITTING OUR STRIDE. NCATS MISSION IS TO TURN BASIC SCIENCE OBSERVATIONS INTO HEALTH SOLUTIONS. TRANSLATIONAL SCIENCE IS OUR SUPER POWER FROM PRE-CLINICAL DRUG DEVELOPMENT TO RESEARCH NCATS LOOKS TO PROVIDE END TO END SOLUTIONS AT A MORE THAN ONE DISEASE AT A TIME PACE. FINDING WHAT'S COMMON TO MAKE TRANSFORMATIONAL CHANGE AND IMPROVING HEALTH. I WELCOME TO YOU OUR ANNIVERSARY CELEBRATION. IT'S NOT REALLY ABOUT CELEBRATING THE PAST IT'S ABOUT CELEBRATING THE FUTURE AND THE REASON WE'RE HERE. WE'RE GOING TO BUILD THE FUTURE TECHNOLOGY. TODAY WE'LL HEAR ABOUT MAJOR SUCCESSES THAT VALIDATE OUR APPROACH AND THROUGHOUT THE TRANSLATIONAL PIPELINE THERE'S TWISTS AND BOTTLENECKS AND EVEN DEAD ENDS FOR EVERY 10 ADVANCES AND TO CHANGE THESE ODDS AND WE NEED ALL OF YOU TO HELP US REENGINEER THE TRANSLATIONAL PIPELINE. THERE'S A LACK OF TREATMENT FOR THOSE WHO NEED THEM AND CONSIDER HOW TO HELP. HOW CAN YOU IMPROVE THE SUCCESS RATES OF THE PRE-CLINICAL AND CLINICAL TRANSITION THAT CAN CHIP AWAY AT THE 90% FAILURE RATE? HOW CAN YOU HELP INCREASE THE DIVERSITY OF PARTICIPANTS IN CLINICAL RESEARCH TO REDUCE INEQUITIES IN CLINICAL OUTCOMES. HOW CAN YOU HELP OPERATIONAL NAD NAD -- INADEQUACIES AND HOW CAN YOU GIVE VOICE TO THE PATIENTS AND MAKE SURE THEY'RE PART OF THE PROCESS. THE PANDEMIC HAS PROVEN WE CAN WORK TOGETHER AND WE CAN DO THAT TO BRING THIS TO ALL AND I'M EXCITED TO GET THE EVENT KICKED OFF. I WANT TO INTRODUCE TWO PEOPLE PIVOTAL IN SHAPING NCATS. WE HAVE MANAGING AT DELOIT CONSULTING AND BRINGS HER ENGAGEMENT TO A BROAD RANGE OF HEALTH INITIATIVE. AS AN ONGOING CHAMPION OF OUR MISSION, MARGARET HAS BEEN INTEGRAL TO SETTING AND KEEPING NCATS ON THE PATH TO SUCCESS. BEFORE I TURN IT OVER TO MARGARET WE HAVE A SPECIAL TREAT, DR. FRANCIS COLLINS NEEDS NO INTRODUCTION BUT I'D BE REMISS TO NOT SAY HOW IMPORTANT HE'S BEEN TO NCATS AND NIH AND MEDICINE AT LARGE. HE'S BEEN OUR FEARLESS DIRECTOR THE PAST THREE PRESIDENTS AND HIS TENURE ENDS THIS MONTH BUT HIS LEGACY WILL BE EVER PRESENT. WHEN COVID-19 EMERGED, FRANCIS CALLED ON NIH AND THE SCIENTIFIC COMMUNITY TO WORK TOGETHER TO MEET THE CHALLENGE AND URGENCY OF THE PANDEMIC. HE CREATED NEW PROGRAMS THAT MOVE WITH UNCHARACTERISTIC SPEED AND SAVE LIVES. AND SINCE THE PANDEMIC HIT, HE'S BEEN IN LOCKSTEP WITH LEADERS WITHIN THE NIH AND ACROSS THE U.S. GOVERNMENT AND ACROSS THE BIOMEDICAL ECO SYSTEM READY TO JUMP IN AND UNSTICK PROBLEMS AND CELEBRATE THE BIG AND SMALL WINS WE NEED ALONG THE WAY. WHILE ALSO SHOULDERING THE WEIGHT OF THE CRISIS ITSELF. IN A RECENT INTERVIEW HE WAS ASKED ABOUT A TOP THREE ACCOMPLISHMENTS. AS NIH DIRECTOR. HE NAMED THE BRAIN INITIATIVE, THE "ALL OF US" RESEARCH PROGRAM AND YES, HE NAMED NCATS. AND I WOULD ALSO LIKE TO ADD AND PERHAPS AN UNDER APPRECIATED ACCOMPLISHMENT OF DR. COLLINS IS LITERALLY CHANGING THE FACE OF THE NIH LEADERSHIP. HE HAS INCREASED THE NUMBER OF WOMEN AND PEOPLE OF COLOR IN SENIOR LEADERSHIP POSITIONS ACROSS THE ORGANIZATION AND HE IS LEADING THE NIH STRONGER FOR IT. PLEASE JOIN ME IN WELCOMING TO THE STAGE DR. FRANCIS COLLINS. >> MY GOODNESS, JONI, THIS WAS GENEROUS. IT'S A PLEASURE TO BE PART OF THIS CELEBRATION OF 10 YEARS OF NCATS. MANY TOMORROW TO YOU AND YOUR STAFF AND FOR THE GREAT SCIENCE LINED UP THE AFTERNOON. I THOUGHT I'D SAY A LITTLE BIT HOW NCATS GOT STARTED. I ASSUMED THE ROLE OF NIH DIRECTOR IN AUGUST OF 2009. I LOOKED TO TRY TO SEE WHAT PRIORITIES WOULD MAKE THE MOST SENSE FOR THE WAY BIOMEDICAL RESEARCH WAS AT THAT POINT AND I OUTLINED FIVE AREAS I THOUGHT DESERVED PARTICULAR ATTENTION AND ONE IS TRANSLATION. HOW DO WE TAKE THE SCIENCE POURING OUT OF LABORATORIES IN TERMS OF BASIC UNDERSTANDING OF HOW LIFE WORKS AND HOW DISEASES HAPPEN AND TURN THAT INTO ACTIONS THAT CAN BENEFIT IN THE CLINICAL WITHOUT GETTING HUNG UP OR HELD UP THERE WERE AMAZING SCIENTIFIC DISCOVERIES ON THE OTHER SIDE WERE THE APPLICATION OF THOSE DISCOVERIES AND ARGUED WE NEEDED TO BUILD A BRIDGE ACROSS THAT SPACE IN ORDER TO MAKE THIS HAPPEN MORE EFFECTIVELY. NOT THAT WE WERE TRYING TO REPLICATE WAS ALREADY DOING BUT RECOGNIZING AN AWFUL LOT OF DISCOVERIES REMAIN RISKY AND NEED TO BE DE RISKED AND OPTIMIZE STRATEGIES TO BE GENERALIZED TO LOTS OF APPLICATIONS INSTEAD OF THE USUAL ONE-OFF APPROACH TO IDENTIFYING A THERAPEUTIC OR DEVICE. THAT WAS A BIG CHALLENGE. IT WAS NOT IMMEDIATELY EMBRACED BY THE SCIENTIFIC COMMUNITY BUT WE NEEDED ANOTHER PART OF NIH TO DO THIS AND ASKS THE MSRB IN 2010 TO LOOK AT THE SITUATION AND WHAT THEY WOULD SAY ABOUT BETTER WAYS TO SUPPORT TRANSLATIONAL SCIENCE AND BY DECEMBER OF 2010 THEY RECOMMENDED WE NEEDED A NEW CENTER THAT NEEDED TO BE CREATED. THAT BECAME NCATS BUT WANTED IT TRULY EMBRACED ACROSS NIH AND SET UP A WORKING GROUP OF MY INSTITUTE AND CENTER DIRECTORS SEEING WHAT THEY THOUGHT INCLUDING PEOPLE LIKE CAROL BAUPIS AND TONY FA FAUCI AND THE DIRECTOR OF THE NATIONAL INSTITUTES OF HEALTH AND CONCLUDED THIS WAS WORTH DOING AND TO MAKE SURE WE HEARD FROM THE EXTERNAL COMMUNITY AS WELL IT WAS NECESSARY AND IMPORTANT TO BASICALLY CONVENE A DIRECTOR TO THE WORKING GROUP WHERE I TURN WITH BIG COMPLICATED ISSUES CHAIRED BY MARIA FERER AND YOU MAY KNOW NOT LONG THAT SHE CAME TO BE THE PRESIDENT AND CEO OF THE FOUNDATION FOR NIH BUT THAT WAS A VERY DISTINGUISHED GROUPS OF EXPERTS AND CAME TO THE CONCLUSION AND CATHY HUDSON THE DEPUTY OF MINE THAT THE POINT PULLING A LOT OF THE WAYS IN WHICH THIS COULD HAPPEN. IT ALL SOUNDS FINE BUT YOU DON'T GET TO START A NEW CENTER AT NIH UNLESS CONGRESS GOES ALONG WITH IT AND THAT WASN'T AN EASY ONE EITHER. AND ON THE SENATE SIDE WE HAD GOOD ENCOURAGEMENT BY SENATOR HARRY REID. ON THE HOUSE SIDE IT WAS UP AND DOWN AND I WAS REFLECTING ON THIS LAST NIGHT AND MIKE MILKEN AND I RECALLING A TIME WHERE NOT BY COINCIDENCE I WAS DOWN VISITING THE HILL WHEN MIKE WAS MEETING WITH ERIC CANTOR AT THE TIME THE MAJORITY LEADER FOR TO THE REPUBLICANS IN THE HOUSE AND CANTOR WAS TRYING TO THINK HE WANTED TO HEAR MORE ABOUT THIS AND BY SUDDEN MAGIC I SHOWED UP IN THE LEADERS OFFICE AND ERIC AND MIKE AND I HAD A DEEP CONVERSATION ABOUT NCATS AND I THINK THAT WAS A TURNING POINT WHERE HE AGREED IT OUGHT TO HAPPEN. SO ON DECEMBER 23, JUST TWO DAYS BEFORE CHRISTMAS, 2011, NCATS WAS ESTABLISHED AS PART OF THE APPROPRIATIONS ACT. THOSE WHO ARE STRUGGLING WITH APPROPRIATIONS THIS YEAR WILL BE FAMILIAR WITH THE FACT THOSE THINGS OFTEN HAPPEN BEFORE THE HOLIDAY THOUGH IN THIS INSTANCE THIS YEAR IT WILL BE LATER SOME TIME IN FEBRUARY. AND TOM ENSOL WAS NAMED AS THE ACTING DRIRTH. PROBABLY A LOT OF PEOPLE DON'T REMEMBER THAT AND BY THAT SMER AFTER A NATIONAL SEARCH AND CHRIS AUSTIN WAS SWORN IN AS DIRECTOR AND EVERYTHING STARTED TO EVOLVE AND DEVELOP AND THE NCATS STYLE WAS IDENTIFIED. PROJECT WERE CATALYTIC THAT HAD GENERALIZABLE INNOVATIVE THAT WERE COLLABORATIVE AND PATIENT FOCUSSED AND NEEDED TO BE MEASURABLE AS WELL. THAT LED TO AREAS INCLUDING WHERE DISEASES AND WHERE NCATS CONTINUES TO BE A MAJOR LEADER IN THAT SPACE. ALL THE THINGS THAT GOT DEVELOPED AND BIO PRINTING AND GENE THERAPY PROCESSES STROME LINED CLINICAL TRIALS AND THEN COVID CAME ALONG RECENTLY AND NCATS WAS WONDERFULLY POSITIONED TO JUMP IN A VARIETY OF WAYS TO ASSIST IN WHAT I THINK PEOPLE WILL SAY WAS A REMARKABLE RESPONSE OF THE PUBLIC HEALTH COMMUNITY TO A CHALLENGE AND WHEN PEOPLE RIGHT THE STORY IT WILL BE I BRIGHT SPOT AND NCATS WAS IN THE MIDDLE OF THAT WITH A NUMBER OF PROJECTS MAYBE I SHOULD MENTION THE ONE IN A BOLD WAY BRINGING TOGETHER THE MEDICAL RECORDS FROM 3 MILLION INDIVIDUALS WERE POSITIVE FOR COVID-19 TO QUICKLY TRY TO SEE WHAT WE COULD LEARN ABOUT THE PROGRESS OF THIS AND THE RISK FACTORS AND THAT IS M3C WHICH CONTINUES TO BE A MAJOR ASSET. IT'S BEEN AN AMAZING 10 YEARS AND I THINK YOU'RE JUST GETTING STARTED, NCATS, BECAUSE YOU ARE IN A PLACE I THINK THAT WILL CONTINUE TO HAVE OPPORTUNITY TO CAT -- CATALYZE THE DEVELOPMENTS AND YOU CAN ADD AND MAYBE ADD SOME LANES AND COME UP WITH OTHER IDEAS HOW TO ACCELERATE THE PROCESS. YES, AS I COME TO THE END OF MY TENURE OF 12 YEARS THREE MONTHS AND 19 DAYS AS DIRECTOR I'M GETTING ASKED BY PEOPLE WHAT ARE THE THINGS YOU THINK ARE MOST SIGNIFICANT AND YES, I'M GLAD YOU NOTE HAD ONE, NCATS IS CERTAINLY ON THAT SHORT LIST OF SOMETHING I FEEL REALLY MADE A DIFFERENCE AND HOPEFULLY WILL CONTINUE TO DO SO WITH ENDURING BENEFITS OF PATIENTS ALIKE AND IT'S OUR PATIENTS WE NEED TO THINK ABOUT IN EVERYTHING WE DO AND NCATS IS RIGHT IN THAT SPACE. SO MANY THANKS TO THE TIRELESS EFFORTS OF VERY IMPRESSIVE AND LONG LIST OF SCIENTISTS WHO ALWAYS FIGURED OUT WAYS THROUGH COLLABORATION TO DO THINGS THAT OTHERWISE WOULDN'T HAVE HAPPENED AND HAVE A BRIGHT FUTURE AHEAD OF THEM. THANK YOU, ALL. IT'S GREAT TO BE PART OF THE PARTY. >> EXCELLENT, FRANCIS, THIS IS MARGARET. CAN YOU HEAR ME? >> I CAN HEAR YOU. SORRY YOUR VIDEO'S NOT LINKING UP BUT I CAN HEAR YOU JUST FINE. >> I'M EXCITED ABOUT THIS BRIEF SESSION WE GET TO HAVE. WE'RE GOING TO MAKE IT COUNT. I WANT YOU TO METAPHORICALLY PUT ON YOUR MOTORCYCLE HELMET SO WE CAN GO FAST, OKAY. >> OKAY. >> WHAT IS IT LIKE AS YOU ENTER INTO THIS PHASE OF YOUR NIH DIRECTORSHIP AND GET TO REFLECT ON THE VAST ACCOMPLISHMENTS WHICH MUST FEEL AMAZING. WHAT'S GOING ON FOR YOU? I KNOW YOU'RE BUSY TRYING TO WRAP THINGS UP. IT HAS GIVEN ME A CHANCE AS PEOPLE ASK FOR REFLECTIONS TO THINK BACK OVER THE 10 YEARS. IT'S NOT SOMETHING I NECESSARILY DO THAT MUCH BECAUSE IT'S ALWAYS SO MUCH IN FRONT OF YOU IN THIS JOB IT'S HARD TO SPEND MUCH TIME ON REFLECTING. THE NIH DIRECTOR HAS THIS AMAZING ABILITY TO CONVENE PEOPLE AND ESTABLISH COLLABORATIONS, GET ALL THE INSTITUTE DIRECTORS ABOUT SOMETHING AND IF YOU HAVE A VISION AND CAN GET OTHER PEOPLE TO SEE IT AMAZING THINGS CAN HAPPEN. THAT'S WHAT NCATS BECAME WAY BACK 10 YEARS AGO AND CONTINUES TO BE TODAY. OTHER THINGS LIKE THAT THE BRAIN INITIATIVE, THE PRECISION MEDICINE INITIATIVE WITH ALL OF US, MORE RECENTLY THE HEAL INITIATIVE ON OPIOIDS AND CHRONIC PAIN AND EVERYTHING WE'VE HAD TO DO FOR COVID-19 THE ACTIVE PUBLIC-PRIVATE PARTNERSHIP AND DIAGNOSTICS. THEY'RE EXHILARATING OPPORTUNITIES TO BRING PEOPLE TOGETHER AND DO THINGS WE OTHERWISE WOULDN'T HAVE BEEN ABLE TO ACCOMPLISH NOT IN THE TIMETABLES NEEDED. >> NCATS WAS EARLY IN YOUR NIH DIRECTOR TENURE. WHAT DID YOU LEARN THROUGH THE PROCESS OF CONCEPTUALIZING IT AND GETTING IT CREATE AND STOOD UP THAT SERVED YOU WELL AS YOU CREATE AND EXECUTED ON SO MANY INITIATIVES SINCE THEN? >> I LEARNED IT'S IMPORTANT TO GET OTHER PEOPLE AS EXCITED ABOUT SOMETHING AS YOU ARE. IT'S NOT GOOD TO RAM AN IDEA THROUGH WHEN NOBODY ELSE SEIZE WHY IT'S GOING TO BE A GOOD THING. I WAS SURE SHORTLY AFTER BECOMING DIRECTOR THIS IS SOMETHING THAT NEEDED TO BE DONE AND I WENT THROUGH THE TIMETABLE OF THE ADVISORY GROUPS WE PULLED TOGETHER THREE OF THEM IN VARIOUS GATHERS OF SENIOR LEADERS TO ULTIMATELY BUILD THE MOMENT UP. I LEARNED IF YOU WANT TO GET SOMETHING DONE IN THIS COMPLICATED ECO SYSTEM YOU WANT TO SURROUND YOURSELVES WITH THOUGHTFUL MOVERS AND SHAKERS AND IF YOU CAN FIGURE OUT A WAY THAT EVERYBODY SHARES THAT VISION THEN IT WILL HAPPEN. THE OTHER THING I LEARNED SI NEEDED TO SURROUND MYSELF WITH A LOT OF SMART PEOPLE WHO ARE READY TO TELL ME WHEN I WAS ABOUT TO MAKE A MISTAKE. THAT'S SOMETHING EVERY LEADER NEEDS TO KNOW THAT EVERYONE AROUND YOU HAS PERMISSION TO SAY, BOSS, THAT'S A BAD IDEA. I'M GLAD THE DYNAMIC FOR ME THAT EXISTED AT NIH ALWAYS GAVE PEOPLE TO PERMISSION TO SEEK WHAT THEY THOUGHT THE TRUTH AND THOUGH IT WASN'T WHAT THE DIRECTOR WANTED TO HEAR IT'S SOMETHING THE DIRECTOR NEEDED TO HEAR. >> I KNOW YOU'RE MUSICALLY DESIGNED AND SO WHICH ARTIST MOST REMINDS OF NCATS AND WHY? >> THERE'S A PERFECT ANSWER TO THAT ESPECIALLY BECAUSE I STARTED OUT TELLING YOU ABOUT MY CARTOON ABOUT THE BIG RIVER WITH THE THE LEFT SHORE BEING BASIC SCIENCE AND THE RED BEING CLINICAL APPLICATION WE NEEDED A BRIDGE OVER TROUBLED WATER. IT'S THE THEME SONG FOR NCATS HENCEFOR HENCEFORTH. >> YOU TALKED ABOUT THE PANDEMIC YOU'RE STILL LEADING THROUGH. IT SHOWED US THE URGENCY OF GETTING NEW TREATMENTS AND TRANSACTIONS THROUGH THE PROCESS QUICKLY. TELL US MORE WHY YOU THINK NCATS WAS SO INTEGRAL TO THE NIH RESPONSE? >> IMMEDIATELY NCATS HAD THE CAPABILITY OF PARTICIPATING IN A MAJOR WAY IN SETTING UP CLINICAL TRIALS NETWORK AND NCATS HAD BEEN A LEADER BY SETTING UP THE APPROPRIATE LIBRARIES AND HIGH THROUGHPUT SCREENING TO SEE IF THERE WERE COMPOUNDS FOR ACTIVITY AGAINST THE VIRUS. ON TOP OF THAT AND RECENTLY NCATS HAS BECOME A MAJOR PART OF OUR EFFORTS TO DEVELOP SPECIFIC ANTIVIRALS TO TARGET THE PROTEASE OR RMA POLYMERASE AND THAT GOING FORWARD WILL BE A MAJOR EFFORT NOT JUST FOR COVID-19 BUT FOR FUTURE PANDEMICS AND THIS IS IN CLOSE COLLABORATION TO IDENTIFY SMALL MOLECULE ANTIVIRALS FOR THE SEVEN OR EIGHT MOST LIKELY NEXT PANDEMIC PATHOGENS MOST OF WHICH ARE VIRUSES. LET'S NOT WAIT UNTIL WE HAVE AN OUTBREAK OF SOMETHING AND GO OH, MY GOSH. WE CAN REASONABLY SAY WHAT FAMILIES WILL PARTICIPATE IN THE NEXT OUTBREAK AND WHY DON'T WE GO NOW TO GO AS FAR AS WE CAN DOWN THE ROAD TO WORK TO A THERAPEUTIC ONE THAT HAS BROAD ACTIVITY AGAINST THE FAMILY AS WE'RE NOW TRYING TO COME UP WITH SMALL MOLECULES THAT WOULD ATTACK ALL CORONAVIRUSES AND MAYBE BEGIN TO MAKE SUCCESSES THERE. NCATS BECAUSE OF THEIR CAPABILITY TO DO THIS KIND OF ASSAY DEVELOPMENT AND HIGH THROUGHPUT SCREENS OF LIBRARIES OF HUNDREDS OF THOUSAND OF COMPOUNDS SIN -- IS IN GOOD PLACE TO MAKE A NEXT STEP TOWARDS THIS PANDEMIC AND FUTURE ONES AS WELL. >> FRANCIS, LET'S PIVOT TO THE NEXT NIH DIRECTOR AND LET'S SPEAK TO NCATS AND WHAT IT'S CAPABLE OF AND HOW TO INTEGRATE IT INTO THE NEXT GEN NIH WHAT IT NEEDS TO ADDRESS GOING FORWARD. >> JUST TO BE CLEAR I HAVE NO IDEA WHO THE NEXT NIH DIRECTOR WILL BE. THAT'S A PRESIDENTIAL APPOINTMENT THE WHITE HOUSE OFFICE TAKES CHARGE OF SURVEYING VARIOUS PROFESSIONAL GROUPS. I HAVEN'T EVEN SEEN A LIST OF WHO IS IN CONSIDERATION. I WOULD LOVE TO SEE SOMEBODY WHO HAS A FULL APPRECIATION OF THE AMAZING SWEEP FROM BASIC SCIENCE ALL THE WAY TO CLINICAL TRIALS AND APPLICATIONS AND YOU CAN'T GET ACROSS THAT SWEEP WITHOUT TRANSLATION. I WANT SOMEBODY FULLY AWARE OF THE CHALLENGES AND OPPORTUNITIES IN TRANSLATION AND THEREFORE WOULD SEE NCATS AS A CRITICAL PART OF THE FUTURE SUCCESS OF THE WHOLE NIH AND PROBABLY WOULD COME TO THIS WITH SOME SPECIFIC IDEAS MAYBE ABOUT DISEASES THAT ARE RIGHT FOR THIS OR ABOUT TECHNOLOGIES THAT MIGHT BE A GOOD FIT. IT SEEMS INCREASINGLY AS WE ADAPT TECHNOLOGIES THINGS WE HAVE SEEN IN MY LIFE TIME LIKE GENE EDITING WE NEED TO HAVE THAT ACUTE ATTENTION TO MAKING THE SCIENCE HAP INSTEAD OF HOPING. THAT'S WHY NCATS KICKS IN. ANOTHER EXAMPLE IS NCATS LEADERSHIP ON THE BESPOKE GENE LEADERSHIP CONSORTIUM AND WHAT WE CAN DO FOR DISEASES WHERE WE KNOW THE SPECIFIC MUTATION AND GENE CAUSING THE DISORDER AND THERE'S THOUSANDS OF THOSE CONDITIONS BUT WE THESE TO BRING THEM TO BEAR NOT JUST AT A BUNCH OF ONE-OFFS WHICH WILL TAKE FOREVER, BUT IN A MORE SALABLE WAY AND THE PARTNERSHIP BETWEEN NIH AND A BUNCH OF INDUSTRY PARTNERS IS GOING TO AIM TO DO AND JONI RUTTER HAS BEEN REMARKABLE IN PICKING UP THE MOMENTUM AND OTHERS WANT TO INVEST AND THIS HAS BEEN ANNOUNCED AND IS HAPPENING AND HERE'S ANOTHER GREAT EXAMPLE WHERE NCATS CAN PLAY A ROLE AND A NEW NIH DIRECTOR WOULD BE EXCITE ABOUT THAT CONSORTIUM PART OF THE ACCELERATING MEDICINES PARTNERSHIP. >> AND YOU'RE BRINGING UP THE TRUE INGREDIENT THE CULTURE OF INFORMED, PERSISTENT, PATIENT ENGAGEMENT AND I THINK IT'S TRUE BASICALLY ON ANYTHING YOU TALKED ABOUT THEY'RE DOING IT WITH THE PATIENT IN MIND AND BRINGING FORWARD SOME BEST PRACTICES. I KNOW IN YOUR FUTURE WORK YOU INTEND TO GET BACK TO BUSINESS IN SOLVING PATIENT PROBLEMS. DO YOU SEE NCATS COMING TO PLAY WITH THE RESEARCH YOU'LL BE LEADING? >> I HOPE SO. I'VE HAD A GRADUATE STUDENT SPEND TIME AT NCATS WORKING ON THE DEVELOPMENT OF AN ASSAY THAT TURNED OUT QUITE WELL. IF YOU WANT MY STRONGEST EVIDENCE OF ENDORSEMENT FOR NCATS IT'S A GREAT PLACE FOR TRAINEES, THIS PAST SUMMER MY GRANDSON WHO IS ABOUT TO BE A FRESHMAN AT HARVARD I SAID IF YOU WANT TO LEARN SOMETHING DO AN INTERNSHIP AT NCATS AND HE DID AND THOUGH IT HAD TO BE VIRTUAL BECAUSE OF COVID, HE LEARNED A TON AND REFLECTS POSITIVELY ON THAT WHEN I TALK WITH HIM NOW THAT HE'S INVESTIGATING SCIENCE IN HIS CURRENT ROLE AS A HARVARD FRESHMAN. WHAT A GREAT ENVIRONMENT WAS CREATED ALL THOSE YEARS AGO AND PROVIDES THAT KIND OF OPPORTUNITY. WHAT AM I DOING? I'M GOING BACK TO MY LAB. I'LL BE AT NHGRI UP BUILDING 50. I'VE HAD THIS LAB GOING SINCE I CAME TO NIH IN 1993, 28 YEARS ABOUT 12 PEOPLE WORKING THERE. WE'RE WORKING ON DIABETES TYPE II AND A RARE DISEASE PROGERRIA WHICH MAY BE ONE OF THE FIRST FOR WHICH IT'S POSSIBLE TO AIM FOR A CURE USING INTRAVENOUS OF A GENETIC MUTATION TO COLLECT THE SINGLE NUCLEOTIDE THAT CAUSES THE DISEASE SO I'LL BE DEPENDENT ON PARTNERSHIP WITH NCATS TO DO THAT PART RIGHT AND IT WILL BE FUN TO BE CLOSER TO THE SCIENCE AND MORE ABLE TO CATALYZE THESE INTERACTIONS AND WHAT YOU SAID ABOUT NCATS IS IT'S A PLACE WHERE PATIENT NUMBER ONE IS CRITICAL. THAT MUST BE SUSTAIN. THE OTHER THING NCATS DOES GREAT IS COLLABORATION AND WHERE THE EXCITEMENT HAPPENS AND WHY NCATS HAS MADE SO MANY ADVANCES POSSIBLE BECAUSE OF THAT COLLABORATIVE APPROACH. >> WELL, FRANCIS, I HAVE TO WRAP AND IN A TEAM SUPPORT ASPECT AND ALL THE STAFF AND LEADERS OF NCATS BELIEVE IN THE TEAM SPORT AND I WANT TO MAKE SURE YOU HAVE FUN WHEN YOU STEP DOWN SO WE'LL BE CROWD SOURCING A NETFLIX PODCAST, BOOK LIST SO YOU CAN HAVE SOMETHING TO DO BESIDES WORK. HOPEFULLY YOU'LL BE PLAYING A LOT OF SONGS AND RIDING YOUR BIKE GETTING CREATIVE AND THANK YOU FOR YOUR LEADERSHIP AND ACCOMPLISHMENTS. I THINK YOU'VE BEEN SELFLESS IN THE COURSE OF THE PANDEMIC AND FOR ME PERSONALLY IT WAS AN HONOR TO WORK WITH YOU AND YOU'RE AN EXTRAORDINARY HUMAN BEING AND GLAD YOU WERE PUT HERE IN THIS TIME AND THIS PLACE AND THANK YOU FOR ALL YOU BROUGHT FORWARD IN TERMS OF THE CREATION OF NCATS. I'LL PASS THE BATON BUT WHAT A PLEASURE ONCE AGAIN TO MODERATE YOU. >> IT'S BEEN A JOY WORKING WITH YOU AND NOW AT DELOIT. IT'S BEEN AMAZING THE KINDS OF THINGS YOU'VE BEEN ABLE TO CATALYZE AND THANK YOU FOR BEING THE EMCEE TODAY AND HAPPY BIRTHDAY NCATS. >> HAPPY BIRTHDAY NCATS. >> HELLO, EVERYONE. I'M PLEASED TO HONOR THE FIRST SESSION FRE TRANSFORMATIONAL POWER OF DATA. I'M CHRISTINE CUTILLO IN THE OFFICE OF THE DIRECTOR OF NCATS. IN THIS SESSION OUR THREE SPEAKERS WILL DISCUSS HOW DATA AND THE PLATFORMS TO ACCESS AND ANALYZE THOSE DATA CAN HELP OVERCOME LARGE SCALE PUBLIC HEALTH CHALLENGES. WHILE THERE'S GREAT POTENTIAL, CHALLENGES ALSO STILL REMAIN IN REGARDS TO ACCESSIBILITY, AVAILABILITY, ETHICS, INNER OPERABILITY AND TRANSPARENCY TO NAME A FEW. AT NCATS WE THINK ABOUT THE BROAD AREA OF DATA SCIENCE THROUGH BOTH THE SCIENTIFIC AND TECHNICAL LENS. THIS COMBINATION IS KEY TO OUR STRENGTH. DATA SCIENTIST AND ENGINEERING AND TRANSLATIONAL RESEARCHERS AND DOMAIN EXPERTS AMONG OTHERS. COLLABORATING AND WORKING ACROSS THE FULL LIFE CYCLE OF A GIVEN EFFORT TO DRIVE HIGH IMPACT INTERVENTIONS FOR PATIENTS WHICH IS THE REASON BEHIND OUR WORK AND KEY TO ADVANCING TRANSLATIONAL SCIENCE. NEXT YOU'LL HEAR ABOUT EFFORTS BY NCATS AND OUR COLLABORATORS AS THEY RELATE TO PATIENT IMPACT AREAS. NOW, ON TO OUR ESTEEMED SPEAKERS. ANNE PARISER IS WITHIN NCATS. ORDR IS DEDICATED TO ACCELERATING RARE DISEASES RESEARCH TO BENEFIT PATIENTS AS PROGRAMS THROUGH GUARD AND THE TOOL KIT FOR PATIENT FOCUSSED THERAPY DEVELOPMENT AMONG MANY OTHERS. PRIOR TO JOINING NCATS IN 2017, ANNE WORKED FOR 16 YEARS AT THE FDA CENTER FOR DRUG VACCINATION AND RESEARCH WHERE SHE FOUNDED THE RARE DISEASES PROGRAM IN CEDAR'S OFFICE OF NEW DRUGS IN 2010. SHE HAS 20 YEARS OF EXPERIENCE IN RARE DISEASE RESEARCH AND HER RESEARCH INTEREST INCLUDES MANY DISEASES AT A TIME APPROACHES SOME SHE'LL DISCUSS SHORTLY. THE SECOND SPEAKER IS MATT MIGHT AT THE UNIVERSITY OF ALABAMA BIRMINGHAM SINCE 2017 WHERE HE IS THE ENDOWED CHAIR OF PERSONALIZED MEDICINE AND PROFESSOR OF INTERNAL MEDICINE AND COMPUTER SCIENCE. FROM 2016 TO 2018 HE WAYS STRATEGIST IN THE EXECUTIVE OFFICE OF THE PRESIDENT IN THE WHITE HOUSE AND A SENIOR LECTURER AT HARVARD MEDICAL SCHOOL AND SCIENTIFIC ADVISER AT PARANOMICS AND REMAINS A BOARD MEMBER SINCE 2018. THE THIRD SPEAKER IS MELISSA HAENDEL. THE DIRECTOR OF THE CENTER FOR DATA TO HEALTH. HER BACKGROUND IS IN MOLECULAR GENETICS AND DEVELOPMENT BIOLOGY AND TRANSLATIONAL INFORMATICS WITH AN FOCUS ON OPEN SCIENCE AND SEMANTIC ENGINEERING AND HER VISION TO LEAD SYSTEMS AND RESEARCH AND PATIENT GENERATED DATA THROUGH DATA INTEGRATION TECHNOLOGIES AND CAPTURE STRATEGIES AND LEADS AND PARTICIPATES IN INTERNATIONAL STANDARD ORGANIZATION TO IMPROVE DATA SHARING AND UTILITY WORLDWIDE. NOW TO ANNE PARISER TO START THE SESSION. THANK YOU. >> NEW, CHRISTINE. I WORK IN THE OFFICE OF RARE DISEASES RESEARCH AND RARE DISEASES IS A BIT OF A MISNOMER AND COLLECTIVELY THERE'S ABOUT 10,000 OR SO DIFFERENT RARE DISEASES AND APPROXIMATELY 25 TO 30 MILLION RARE DISEASE PATIENTS IN THE UNITED STATES. DATA IS VERY IMPORTANT TO US. WE HAVE LIMITED INFORMATION ON THE INDIVIDUAL DISORDERS. DATA IS VERY APPRECIATION DID -- PRECIOUS AND TRYING TO GIVE EXAMPLES OF EFFORTS UNDERTAKEN USING THE POWER OF DATA TO TRY TO HELP US UNDERSTAND OUR PATIENTS NEEDS AS WELL AS IDENTIFYING THE AREAS FOR CONTINUING RESEARCH. THE FIRST EXAMPLE IS SOMETHING WE CALL THE IDEAS INITIATIVE. THIS WAS A PILOT STUDY THAT WE CONDUCTED WHERE WE ANALYZED HEALTH CARE SYSTEM DATA ON RARE DISEASE PATIENTS AND WE HAD SEVERAL GOALS HERE. ONE WAS TO TRY TO BETTER QUANTIFY AS THE IMPACT OF RARE DISEASES ON PATIENTS THEMSELVES ALSO HEALTH CARE SYSTEMS AND UTILIZATION AND COST AND LOOKING FOR SIGNALS WITHIN THE DATA THAT MIGHT HELP US IDENTIFY PATIENTS FAST FASTER AND TO BETTER UNDERSTAND AREAS TO INTERVENE. THIS WAS A COLLABORATION LIKE EVERYTHING NCATS DOES. SEVERAL EXTERNAL STAKEHOLDERS. RARE DISEASE NOT IN FACT RARE COLLECTIVELY. THEY'RE QUITE COMMON SO THIS IS A BIG PUBLIC HEALTH ISSUE AND WHAT IS NO SURPRISE AT ALL TO THE RARE DISEASE COMMUNITY IS RARE DISEASES ARE VERY COSTLY. THEY'RE COSTLY IN MATERIALS OF DOLLARS BUT COSTLY IN TERMS OF WHAT THE BURDENS CARRIED BY PATIENTS. MOST RARE DISEASES ARE SERIOUS. AND VERY IMPORTANTLY IS WE SAW SIGNALS THESE WERE ACTIONABLE IN TERMS OF DIAGNOSIS AND WHERE WE CAN FOCUS OUR RESEARCH. THIS IS AN OPEN ACCESS PAPER AND HOPE YOU'LL LOOK AT THAT AND WELCOME COMMENTS. THERE WERE SOME EXTERNAL STAKEHOLDERS DOING SIMILAR PROJECTS AND DATA IS MOST HELPFUL AND MOST TRANSFORMATIVE WHEN IT IS SHARED. WE ARE NOW REACHING OUT BEYOND NCATS AND OTHER AREAS TO MAKE THE DATA WORK. THE NEXT EXAMPLE I'D LIKE TO SHOW YOU IS A LONG STANDING PROGRAM AT NIH CALLED THE GENETICS AND RARE DISEASES INFORMATION CENTER OR GARD. GARD HAS BEEN AROUND SINCE 2003. AND TRYING TO PROVIDE INFORMATION ABOUT 6500 OR SO RARE DISEASES. THIS IS THE GARD 1.0 SITE. YOU SEE THE PURPLE BOX FOR THE GARD 2.0 SITE AND WE HAVE GOALS HERE AS WELL. ONE WAS TO TRY TO TO MAKE IT MORE USEFUL TO PATIENTS. WE WERE TRYING TO PROVIDE INFORMATION ACTIONABLE AND IT COULD BE PRINTED OFF AND ALSO A LEARNING SITE. WHAT WE'D ENCOURAGE YOU TO DO IS IF YOU PRESS THE PURPLE BUTTON YOU CAN GIVE FEEDBACK ON THE SITE. IT'S IMPORTANT BECAUSE WE WANT GARD TO BE USER CENTERED. THAT'S IS WE'LL CONTINUOUSLY UPGRADE AND WORK ON GARD BASED ON YOUR FEEDBACK AND WHAT YOU'RE TELLING US IS USEFUL TO YOU. AND WE'RE TRYING TO TAKE THE PATHWAYS OF INFORMATION AND TRY TO HARVEST DATA AND BETTER UNDERSTAND RARE DISEASES AND SHARE IT. THIS IS A WORK IN PROGRESS AND COME TO THE SITE AND GIVE US FEEDBACK. WE'D WELCOME THAT. AND THE LAST EXAMPLE I'D LIKE TO GIVE YOU IS FROM THE RARE DISEASES CLINICAL RARE DISEASES NETWORK ANOTHER LONG STANDING NCATS PROGRAM AND THIS IS THE NETWORK IS A COLLECTION OF 20 RESEARCH CONSORTIA ABLE TO STUDY ABOUT 250 RARE DISEASES AND TRY TO PROMOTE CLINICAL TRIAL READINESS AND PATIENT IN RARE DISEASE RESEARCH. STARTING LAST MAY DURING THE PANDEMIC AND WE WERE TRYING TO BETTER UNDERSTAND IMPACT ON FAMILIES AND THROUGH GOOD QUALITY DATA. WHAT WE'VE DONE IS THE RESEARCHERS SENT EIGHT SURVEY AND WE GOT SEVERAL THOUSAND RESPONSES TRYING TO UNDERSTAND EXACTLY HOW COVID WAS IMPACTING OUR FAMILIES AND PATIENTS. WE HARVESTED QUITE A BIT OF DATA FROM THIS AND IT'S STILL BEING ANALYZED AND WE THANK EVERYBODY WHO PARTICIPATED IN THIS. AND WE ACTUALLY LEARNED A LOT FROM THIS STUDY. WE WILL CONTINUE TO ANALYZE THIS AND CONTINUE TO COLLECT DATA BUT THESE WERE JUST A FEW EXAMPLES OF HOW THIS IS USEFUL. SO THOSE ARE THE EXAMPLES I WANTED TO SHARE. I KNOW WE'LL HAVE A DISCUSSION ABOUT THIS BUT AT THIS POINT I'D LIKE TO TURN IT OVER TO MATT WHO I KNOW HAS ADDITIONAL EXAMPLES. THANK YOU. >> ALL RIGHT, WELL, AS MY FORMER LIFE AND PRESENT LIFE I'VE COME TO APPRECIATE THE TRANSFORMATIONAL POWER OF DATA IN CONTEXT AS FAR AS PERSONALIZ PERSONALIZED DIABETIC CARE AND BY ADOPTED FIELD OF PRECISION MEDICINE IS IN ESSENCE ONE THAT BECOMES WHEN WE TEND TO OPTIMIZE THE CARE OF THE PATIENT WITH RESPECT TO ALL AVAILABLE DATA AND WITH NCATS DATA HAS BEEN A PIVOTAL FORCE FROM THE FLAGSHIP TRANSLATOR PROGRAM TO EFFORTS AT DRUG REPURPOSING FOR RARE DISORDERS AND THE EXPANSIVE RESEARCH NETWORK GENERATING TREASURE TROVES OF DATA. FOR PURPOSES OF THIS TALK WE CAN LOOK AT THE DATA ON PATIENTS ON BIOMEDICAL RESEARCH ITSELF AND MY REMARKS TO THE BIOMEDICAL DATA TRANSLATOR. THE GRAND CHALLENGE OF OPTIMIZING HEALTH WITH RESPECT TO DATA HAS TWO MAJOR SUB CHALLENGES. THE ONE IS MANAGE THE QUANTITIES OF DATA AVAILABLE IN CONJUNCTION WITH THE WEALTH OF DATA AND KNOWLEDGE IN BIOMEDICAL RESEARCH AS A WHOLE. THE SECOND CHALLENGE IS THE COMPLEXITY OF ANALYZING AND REASONING OF DATA AND IT TURNS OUT THE BIOMEDICAL TRANSLATOR PROGRAM ADDRESSES BOTH CHALLENGES DIRECTLY. WITH THE TIME THAT'S LEFT IT'S IMPORTANT TO MAKE IT CLEAR HOW IT POTENTIATES DATA INTO ACTION AND ACCELERATES SCIENCE IN THE SERVICE OF PATIENTS. SO MAY BE THE BEST WAY TO UNDERSTAND THE FULL AMBITION IS TO IMAGINE YOU HAVE A SMART FRIEND AND READS EVERYTHING AND DISTILLS EVERYTHING IN BIOMEDICAL RESEARCH AND YOU CAN ASK THAT FRIEND QUESTION LIKE WHAT ARE WAYS TO TREAT DISEASE X OR LOW A LOW-LEVEL QUESTION LIKE WHAT ARE POSSIBLE INHIBITORS FOR Y AND TRANSLATORS CREATING THAT FRIEND AND BRINGING HER MORE TO LIFE EVERY DAY AND THIS TRANSLATOR IS ALREADY SAVING LIVES AND ACCELERATING SCIENCE. FOR ONE EXAMPLE A COUPLE YEARS AGO A MOTHER REACHED OUT TO ME AND ASKED FOR HELP FOR HER SON SUFFERING FROM AUTISM DRIVEN BY A MUTATION IN A GENE CALLED ADNP AND WE JUST CREATED A WAY TO ASK CERTAIN QUESTIONS OF THIS REMARKABLE KNOWLEDGE BASE. UNDERSTANDING THAT IN THIS CASE WE WERE DEALING WITH UNDER ACTIVITY IN THE GENE ADNP AND ASKED TRANSLATOR WHETHER IT WOULD FIND DRUGS TO ENHANCE THIS ACTIVITY AND IT DID AND IT SUGGESTED KETAMINE. ALL THE EVIDENCE THAT TRANSLATOR GENERATED AFTER MONTHS OF PURSUING THAT LEAD ARMED WITH ALL THIS DATA THIS MOM CONVINCED THE FDA TO LAUNCH CLINICAL TRIALS AND IT WORKED. ACROSS A BROAD SPECTRUM OF END POINTS EVERY CHILD SHOWED IMPROVEMENT AND TRANSLATOR'S CORE PREDICTION THAT KETAMINE COULD RESTORE ACTIVITY WAS VALIDATED DIRECTLY IN THE TISSUES OF THE PATIENTS THEMSELVES. THIS IS AS CLEAR AN EXAMPLE AS ONE CAN GET TRANSLATING SCIENCE IN THE CARE OF PATIENTS. IT'S A GROWING NUMBER OF STORY WE'VE BEGUN TO WEAVE USING THE POWER OF DATA. MY REASON FROM SWITCHING FROM COMPUTER SCIENCE TO MEDICINE IS MY SON. HE WAS THE FIRST CASE OF AN NY1 DEFICIENCY AND TRANSLATOR MADE MORE THAN ONE IMPACT INCLUDING SEVERAL REPURPOSED TREATMENTS THAT BENEFITTED HIM BUT I CAN PICK OUT ONE EPISODE IN WHICH NO MORE DRAMATIC MEASURE OF THE IMPACT WHEN HE WENT INTO SEPTIC SHOCK IN MAY 2019 AND SPENT FIVE WEEKS IN THE ICU AND WAS SUFFERING FROM A DEVASTATING INFECTION AND HAD ALL THE TRADITIONAL METHODS AND COULD NOT FIGURE OUT WHAT PATHOGEN WAS RESPONSIBLE AND I TAPPED INTO TRANSLATOR'S KNOWLEDGE BASE IN A WAY I HADN'T BEFORE INCLUDING HEAVY USE OF DATA SETS PROVIDED BY THE SPEAKER FOLLOWING ME, MELISSA, TO CRAFT A DIAGNOSTIC MODE AND CONSIDERED THEM AGAINST THE BACKDROP OF BIOMEDICAL FACTS AND SPIT OUT POSSIBLE DISEASES. REMARKABLY IT WORKED GETTING US DOWN TO A SHORT LIST OF EIGHT POSSIBLE PATHOGENS AND FEEDING IN METAGENOMIC DATA TO A VERY UNUSUAL PATHOGEN AND FROM THIS IT SUGGESTED THE RIGHT ANTIBIOTIC TO TREAT AND AFTER FOUR WEEKS HE IMPROVED OVER THE FIFTH AND FINAL WEEK AND WENT HOME. I REMEMBER EVERY DAY WAS A BONUS DAY AND EVERY ONE OF THOSE BONUS DAYS WAS OWED TO TRANSLATOR. HE PASSED AWAY SUDDENLY A YEAR AGO DURING THE PANDEMIC. I DON'T KNOW HOW TO PUT A VALUE ON THE BONUS DAYS BUT IT WAS MORE DAYS OF A PARENT'S LOVE THAT WOULD NOT HAVE OTHERWISE BEEN THANKS TO TRANSLATOR, THANKS TO NCATS AND THANKS TO EACH AND EVERY ONE OF YOU THAT WORKS AT NCATS THAT BROUGHT THIS MIRACLE FACTOR TO LIFE OVER THE LAST 10 YEARS. AS I THINK ABOUT THE FUTURE OF NCATS AND THINK OF TRANSLATOR THAT ARE POWERFUL AND IT'S STILL IN ITS INFANCY AND ACHIEVED ONLY A FRACTION OF ITS FULL POTENTIAL AND LEVERAGING POWER OF DATA TO ACCELERATE TRANSLATIONAL SIGN AND HELP PATIENTS IN THE PROCESS. EACH YEAR IT'S KNOWLEDGE BASE CONTINUES TO GROW AND REASONABLE AND CAPABILITY EXTEND IN BREADTH AND DEPTH. WITH ACCESS TO WELL STRUCTURED ELECTRONIC HEALTH RECORDS IT'S NOT HARD TO IMAGINE WHERE IT'S ASSISTING EVERY POSITION AT EVERY BEDSIDE FOR EVERY PATIENT AND AS SCIENTIFIC DATA AND KNOWLEDGE BECOMES STANDARDIZED AND STRUCTURE IT'S NOT HARD TO IMAGINE IT AS A CO-PILOT FOR RESEARCHERS SYSTEMATICALLY EXPANDING HUMAN KNOWLEDGE AS A WHOLE AND WITH THAT I'D LOVE TO TURN IT OVER TO MELISSA. >> THANK YOU, IT'S A GREAT PLEASURE TO BE HERE AND PARTICIPATING IN OUR LONG STANDING COLLABORATION. IT TAKES A VILLAGE. TODAY I'LL TALK ABOUT I WORK ON COVID AND HOW NCATS HAS LED A SUBSTANTIAL EFFORT AS MENTIONED BY DR. FRANCIS COLLINS IN THE EARLIER SESSION. SO SIMILAR TO RARE DISEASES, UNDERSTANDING A NEW DISEASE IS VERY CHALLENGING. WE NECESSARY HAVE TO HAVE A LOT OF DATA TO UNDERSTAND THE SPECTRUM OF SYMPTOMS AND UNDERSTANDING OF UNDERLYING MECHANISMS AND AN UNDERSTANDING OF WHAT DRUGS MAY EXACERBATE OR AMELIORATE THE CONDITION AND THAT'S TRUE FOR ALL RARE DISEASES BUT ESPECIALLY TRUE IN THE CASE OF COVID-19. AND N3C WAS LAUNCHED BY NCATS UNDER THE LEADERSHIP OF KEN GERSING WITH JOHNS HOPKINS AND MYSELF AS A WAY TO COORDINATE THE CTSA PROGRAM SITE 60 OF THE ACADEMIC MEDICAL CENTERS IN THE UNITED STATES TO UNDERSTAND HOW TO USE ELECTRONIC HEALTH RECORD DATA TO HELP PROVIDE THE FOUNDATION OF DATA TO ASSESS WHAT ARE THE COMORBIDITIES, WHO IS HAVING THE MOST SEVERE CASES OF COVID AND HOW TO PREVENT TOGETHER AND SEVERE OUTCOMES FROM COVID. IT'S AN EXAMPLE OF THE TRANSFORMATIVE POWER OF DATA NOT ONLY IN THE FACE OF THE NEW DISEASE BUT THE WAY IN WHICH WE DO SCIENCE. THIS EFFORT IS ONE IN WHICH IT TAKES A VILLAGE. I WANT TO GIVE EXAMPLES OF THE TYPES OF VILLAGES THAT IT TOOK IN ORDER TO CREATE SUCH AN INCREDIBLE RESOURCE. THE FIRST THING IS PEOPLE SAY IF YOU'VE SEEN ONE ELECTRONIC HEALTH RECORD SYSTEM YOU'VE SEEN ONE AND THE DATA PRESENT WITHIN OUR HOSPITAL SYSTEMS NOT DESIGNED FOR INTEROPERABILITY. WHILE WE MAY BE ABLE TO DO RESEARCH WORK OR COVID ANALYTICS IN HOSPITALS IT'S NOT ENOUGH DATA IN THE FACE OF A NEW OR RARE DISEASE. WE NEED TO SCALE THAT ACROSS INSTITUTIONS. IN ORDER TO DO THAT WE NEED TO THINK ABOUT HOW DO WE FIRST OBTAIN THE DATA AND PUT THEM IN A SECURE LOCATION AND HARMONIZE THEM TO DO MODERN ARTIFICIAL INTELLIGENCE AND MACHINE LEARNING ANALYTICS TO REVEAL THE PATTERNS OF CARE AND OUTCOMES TO HELP PREVENT MORBIDITY AND NEGATIVE OUTCOMES. A LARGE TEAM OF EXPERT IN MANY DIFFERENT COMMON DATA MODELS, MODELS THAT HELP SUPPORT THE STRUCTURING OF DATA HAVE A TRANSFORMATIVE PIPELINE FOR THE FIRST TIME EVER AND NOW THE SYSTEM CONTAINS THE LARGEST PUBLICLY AVAILABLE LIMITED DATA SET IN U.S. HISTORY WITH 67 DIFFERENT INSTITUTIONS ALREADY IN THE DATA SET AND AND IN THE EARLY DAYS THIS TEAM WAS ABLE TO HARMONIZE DATAS TO COLLABORATIVELY PERFORM THE KIND OF ANALYTICS. AND NOT ONLY DID NCATS PROVIDE THE LEADERSHIP AND FUNDING AND COORDINATION TO HELP MAKE THIS POSSIBLE ACROSS THE CTSA PROGRAM BUT LAUNCHED IT AS AN NIH AND NATIONWIDE INITIATIVE PARTNERING WITH THE CENTERS OF TRANSLATIONAL SCIENCE AND RURAL HEALTH CARE NETWORKS TO WORK COLLABORATIVELY TOGETHER WITH INSTITUTIONS AND INVESTIGATORS WHO WEREN'T NECESSARILY AT A CTSA INSTITUTION WITH MANY KUDOS FOR THE VILLAGE CREATED UNDER NCATS SLEERDSHIP. I WANTED TO GIVE A FEW EXAMPLES HOW THE DATA WERE USED. FIRST I SHOULD MENTION ONE OF THE MOST EXCITING THINGS IS ALL THE EXAMPLES ARE FULLY REPRODUCIBLE AND PUBLICLY ACCESSIBLE. IT'S OFTEN THE CASE WE THINK OF CLINICAL DATA SO SENSITIVE IT CANNOT BE SHARED. THERE'S MANY RETRACTIONS FROM CLINICAL RESEARCH STUDIES BECAUSE THE STUDIES ARE NOT TRANSPARENT AND THE DATA'S NOT AVAILABLE. IT'S THE FIRST EFFORT IN WHICH WE CAN DO COLLABORATIVE OPEN SCIENCE ON CLINICALLY SENSITIVE DATA AT SCALE. THE FIRST EXAMPLE ALLOWS THE COLLECTION OF LABORATORY VALUES ON THE FIRST DAY OF ADMISSION FOR COVID POSITIVE PATIENTS TO PREDICT THEIR OUTCOMES BASED ON THE FIRST DAY VALUES. THE EXCITING THING ABOUT THIS ANALYSIS AND RESOURCE IS THAT THESE ARE COMMONLY USED AROUND THE WORLD TO PREDICT THE COVID SEVERITY OUTCOMES OF A PATIENT AND THERE BE HELP MANAGE THEIR CARE BETTER AND MAKE HARD AND IMPORTANT DECISIONS IN RESOURCE ALLOCATION AND THE SECOND IS A DIABETES EXAMPLE WHERE WE KNOW IT'S ONE OF THE COMORBIDITIES MOST STRONGLY ASSOCIATED WITH COVID-19 IN THE UNITED STATES AND DATA IN THE EARLY PANDEMIC SHOWED INDIVIDUALS WITH DIABETES HAVE TWICE THE RISK OF DEATH AND HOSPITALIZATION AND CARE AND THERE WERE MEDICATION LOWERED BLOOD SUGAR AND WERE ABLE TO PUBLISH A MANUSCRIPT TO CHANGE GUIDELINES FOR PATIENTS ON THESE PARTICULAR DRUGS TO PUT THEM ON ONE OF THE ONES SUPPORTING POSITIVE OUTCOMES OPPOSED TO THE ONE THAT SEEMED TO BE ASSOCIATED WITH MORE NEGATIVE OUTCOMES AND IT'S ONLY THROUGH THE TRANSFORMATIONAL POWER OF HAVING SO MANY UNFORTUNATELY, DIABETIC PATIENTS WITH COVID IN OUR SYSTEM WE WERE ABLE TO CREATE THAT ANALYSIS AND CORRELATIONS TO HELP GUIDE CARE. THE LAST EXAMPLE I WANTED TO PROVIDE IS ABOUT A CONDITION WE CALL LONG COVID. SO AS MANY ARE AWARE THERE'S MANY LONG TERM CONSEQUENCES OF COVID AND TRYING TO UNDERSTAND HOW THOSE ARE IN THEMSELVES THEIR OWN KIND OF NEW DISEASE GETS TO THE QUESTION OF HOW DO YOU DEFINE A DISEASE AND HOW MUCH DATA DO YOU ACTUALLY NEED IN ORDER TO DO SO. SOMETHING WE ALSO STRUGGLE WITH IN THE RARE DISEASE COMMUNITY AS WELL. SO WHAT WE WERE ABLE TO DO WAS TO BUILD A MACHINE LEARNING MODEL THAT ALLOWED US TO PREDICT WHICH PATIENTS MAY BE CANDIDATE FOR RECEIVING CARE IN A LONG COVID CLINIC AND WHO HAS LONG COVID IN THE ABSENCE OF A DEFINITION. WE KNOW LONG COVID HAS MANY DIFFERENT SYMPTOMS AND ACTUALLY HAVE DONE RESEARCH IN THE LITERATURE AS WELL AS COLLABORATING WITH THE LARGEST COVID PATIENT GROUP IN THE WORLD THE PATIENT-LED RESEARCH COLLABORATIVE WHO ARE NOT ONLY LONG COVID PATIENTS BUT HAVE WONDERFUL EXPERTISE IN DATA SCIENCE TO UNDERSTAND THE SPECTRUM OF SYMPTOMS THAT LONG COVID PATIENTS EXPERIENCE SUCH AS TROUBLE BREATHING, FATIGUE, INSOMNIA, COGNITIVE IMPAIRMENT AND ONE OF THE BIGGEST CHALLENGES WE HAVE IN CREATING THIS MUCH LEARNING MODEL NOT ALL OF THOSE SYMPTOMS ARE NECESSARILY RECORDED IN THE EHR. SO WORKING TOGETHER WITH THE PATIENT GROUP AND OF THE INSTITUTIONS PARTICIPATING WE CAN CREATE A MORE ROBUST MODEL FOR CHARACTERIZING LONG COVID WHERE WE HAVE HUNDREDS OF PATIENTS THAT MAY BE GOOD CANDIDATES FOR RESEARCH ON LONG COVID. I WAS ASKED ABOUT TO SPEAK ABOUT THE IMPORTANT DATA SETS AND CHALLENGES MOVING FORWARD. ONE OF THE THINGS I THINK WE'VE TOUCHED UPON IN ALL THE TALKS TODAY BUT I THINK HITS HOME IN THE CONTEXT OF COVID IS WE NEED TO PUT THE PATIENT BACK TOGETHER AGAIN AND IN RESEARCH SCIENCE AND CLINICAL RESEARCH WE CAPTURE DATA ABOUT A PATIENT AND CAPTURE GENOMICS AND TAKE SURVEYS AND LABORATORIES COLLECTED FOR CLINICAL RESEARCH STUDIES AND SHIP THEM TO A GENOMIC DATA AND IMAGING REPOSITORY AND CLINICAL EHR REPOSITORY AND IT'S CHALLENGING TO THINK ABOUT HOW TO UNDERSTAND THE PATIENT'S DISEASE TRAJECTORY IN THE FACE OF THESE DIFFERENT DATA ABOUT AN INDIVIDUAL PATIENT REALLY NOT BEING CONNECTED ANYWHERE. ONE OF THE MOST IMPORTANT PAN-NIH INITIATIVES LED BY NCATS IS THE DATA PRESERVINGEST TO ALLOW US TO PUT THE PATIENT BACK TOGETHER AND TAKE THE DATA WHETHER IT'S VIRAL SEQUENCE DATA FROM COVID OR CMS DATA, IMAGING DATA AND OTHER DATA MODALITIES AS WELL AS DUPLICATING PATIENTS WHO MAY HAVE BEEN SEEN AT DIFFERENT INSTITUTIONS TO CREATE THE LONGITUDINAL COMPLETE PICTURE OF THE PATIENT WITH THIS PPRL AND WE NEED TO IMPROVE THE DATA WE'RE COLLECTING ABOUT PATIENTS LIKE SOCIAL DETERMINATES OF HEALTH AND PATIENT-REPORTED INFORMATION BECAUSE WE KNOW MANY OF THE SYMPTOMS REPORTED BY PATIENTS FOR LONG COVID ARE SIMPLY NOT REPRESENTED IN THE EHR. FINDING NOVEL WAYS OF PATIENT-REPORTED INFORMATION AND ALIGNING THOSE IT DATA IN THE HR ARE GOING TO BE PIVOTAL FOR THE FUTURE OF RESEARCH AND THE MOST EXCITING THING IS COMBINING THE DATA FROM TRANSLATOR WING -- WITH OUR WORK AND WE'VE BEEN ABLE TO PUT THIS ARTIFICIAL INTELLIGENCE MECHANISTIC RESOURCES TOGETHER INTO A SECURE LOCATION ALONG SIDE THE ELECTRONIC HEALTH RECORD DATA AND BY USING SOPHISTICATED TECHNOLOGY TOOLS TO ACROSS THE CHASM OF SEMANTIC DESPAIR AND HOW TO RELATE DATA ON THE CLINIC TO BASIC SIDE WE'RE ABLE TO USE THAT MECHANISTIC INFORMATION TOGETHER WITH THE CLINICAL INFORMATION PRESENT WITHIN THE ELECTRONIC HEALTH RECORD. I BELIEVE SIMILAR TO WHAT MATT HAS SUGGESTED THE NCATS DATA TRANSLATOR SHOULD BE AT THE FINGER TIPS OF THE CLINICIAN WHENEVER THEY NEED ACCESS TO THAT KIND OF CHARACTERIZATION FOR EVALUATING CANDIDATE DIAGNOSIS OR THERAPIES AND THAT FOUNDATION OF INTEROPERABILITY BETWEEN THE BASIC RESEARCH AND CLINICAL DATA TO GET US WHERE WE WANT TO GO IN THE NEXT FEW YEARS. A FEW TAKEAWAYS. ONE OF THE MOST IMPORTANT TAKEAWAY IS TEAM SCIENCE. WE HAVE OVER 2500 INVESTIGATORS WORKING IN THE ENCLAVE. IT'S AN UNPRECEDENTED SCALE OF TEAM SCIENCE WE HAVE CLINICIAN SCIENTISTS, INFORMATICIANS, ALL WORKING HAND IN HAND ON PARTICULAR TOPICS WITHIN THE CONTEXT OF THE ENCLAVE. THE OTHER EXCITING THING IS ABOUT ATTRIBUTION. BECAUSE IT TAKES A VILLAGE TO BRING ALL THE DATA TOGETHER AND ANALYZE IT WELL, WE ALSO HAVE BECAUSE OF THE REPRODUCIBILITY AND SECURITY AVAILABLE IN THE ENCLAVE WE HAVE AN UNPRECEDENTED ABILITY TO ATTRIBUTE EVERY PERSON WHO CONTRIBUTES BECAUSE WE KNOW BECAUSE OF THE SECURITY REQUIREMENT WHO HAS CONTRIBUTED TO WHAT AND SO IT MAKES IT A REALLY INVITING WAY TO MAKE SURE WE ARE ATTRIBUTING EVERYBODY IN ALL THE WAYS THEY CONTRIBUTE TO MAKE THIS TYPE OF SCIENCE GO. I THINK THE FUTURE OF NCATS AND HARNESSING THE POWER OF DATA IS ALSO HARNESSING THE POWER OF PEOPLE ASSOCIATED WITH THAT DATA. >> THANK YOU TO OUR THREE SPEAKERS THROUGH HEALTH LITERACY AND CONNECTING DATA TYPES TO ANSWER COMPLEX QUESTIONS AND FRAME THE NEEDS OF PATIENTS THROUGH THE TRANSLATOR PROGRAM. AND FINALLY, WITH THE COLLECTION AND AGGREGATION OF REAL WORLD DATA BY EXTENSIVE COLLABORATION AND INHERENT CHALLENGES THAT REMAIN TO LEVERAGE THIS DATA IN THE FUTURE THROUGH THE N3C INITIATIVE WITH COVID-19 AS THE TIMELY AND URGENT USE CASE. THERE'S AMAZING PROMISE AND KEY CHALLENGE TO NEXT TACKLE. AT NCATS WE'RE LOOKING AT PILLARS IN OUR WORK BEING INNOVATIVE, SUSTAINABLE WHILE ENSURING DIVERSITY, EQUITY AND INCLUSION AND BEING TRANSPARENT, OPEN AND COMMUNICATIVE. ULTIMATELY TO FOSTER IMPROVEMENT TO ADVANCE SCIENTIFIC STEWARDSHIP AND FAIR PRINCIPLES BY MAKING SCIENCE AND DATA OPEN AND ACCESSIBLE. WE ARE EXCITED ABOUT THE FUTURE AND INVITE AND HOPE YOU WILL JOIN US IN HARNESSING THE POTENTIAL POWER OF DATA IN TRANSLATIONAL SCIENCE. THANK YOU ALL VERY MUCH FOR ATTENDING THIS FIRST SESSION AND WE WILL NOW BE MOVING INTO A SHORT FIVE-MINUTE BREAK AND AFTERWARD START WITH THE SECOND SESSION ON ENABLING PROMISING PRE-CLINICAL CANDIDATES. THANK YOU FOR YOUR TIME. >> GOOD AFTERNOON AN AND WELCOME TO SESSION 3 ENABLING PROMISING CLINICAL CANDIDATES. I'M AT THE MILKEN INSTITUTE AND PLEASED TO PARTICIPATE IN THIS 10th YEAR CELEBRATION OF NCATS. DURING THIS SESSION WE'LL HEAR CONSIDERING STORIES ON THE JOURNEYS SCIENTIST HAVE TAKEN TO ARRIVE AT THEIR ROLES IN BRIDGING TRANSLATIONAL AND PRE-CLINICAL RESEARCH USING NEW TOOLS AND METHODS TO SPEED INNOVATION. IMPORTANTLY, THEIR WORK IS DRIVEN BY THE NEEDS OF PATIENTS, FAMILIES AND CAREGIVERS DESPERATELY LOOKING FOR NEW TREATMENTS AND CURES. WE'LL HEAR ABOUT CUTTING TOGETHER TECHNOLOGY INCLUDING INNOVATIVE TECHNOLOGY SUCH AS 3-D TISSUE BIOPRINTING. I'M PLEASED TO WELCOME A GROUP OF AMAZING SCIENTISTS AND LEADERS WE'LL HEAR FROM IN THREE TALKS. THE FIRST DR. DON LOWE. THE DIRECTOR OF THE THERAPEUTIC BRANCH FOR ADVANCING TRANSLATIONAL SCIENCE AND PRE CLINICAL INNOVATION RESPONSIBLE FOR A PROGRAM IN PRECLINICAL V AND THE LEAD OPTIMIZATION TOXICOLOGY AND PHARMACOLOGY, FORMULATION DEVELOPMENT AND PROCESS CHEMISTRY AND NATURAL HISTORY STUDIES AND BIOMARKER DEVELOPMENT. PLEASED TO WELCOME DR. TIM YU AT BOSTON'S CHILDREN'S HOSPITAL AND HARVARD MEDICAL SCHOOL AND LEADS AT THE INTERSECTION OF NEUROBIOLOGY AND USING TOOLS TO UNDERSTAND AND TREAT RARE PEDIA PEDIATRIC DISEASES. WE HAVE A RARE DISEASE DRUG DEVELOPER. ALSO PLEASED TO INTRODUCE DR. MARK FERER. AT NCATS AND LEADS THE MULTI DISCIPLINARY GROUP WITH A VALIDATING AND USING 3-D BIO ENGINEERED TISSUES FOR DISEASE MODELLING AND FINALLY HERE FROM DR. DAN TAGLE AND SERVES AS THE ACTING DEPUTY DIRECTOR AS WELL AS THE ACTING DIRECTOR OF NCATS OFFICE OF BRANCH MANAGEMENT, SCIENTIFIC REVIEW AND EXECUTIVE SECRETARY TO THE NCATS ADVISORY COUNCIL. PLEASED TO HEAR FROM ALL OF YOU AND FIRST FOR TALK ONE TO DON AND SANATH AND TIM. >> AS MANY HAVE SAID THROUGH THE COURSE OF THE DAY WITH JONI AND FRANCIS AND ALL OF US WE'RE ALL ABOUT THE SCIENCE AND ABOUT THE PEOPLE AND THE TEAM WORK WE THOUGHT WE'D CHANGE IT UP AND HAVE US THREE AT THE SAME TIME AND DO A FIRE SIDE CHAT AND GET MORE OF A DISCUSSION TO GIVE EVERYBODY A BETTER IDEA OF HOW IT IS WE INTERACT AND HOW TEAMS WORK TOGETHER. I GOT THE COIN TOSS TO START OFF AND I'LL GIVE A SHORT STORY HOW I GOT HERE AND WHAT WE DO AND HOPE TIM WILL ALSO NOT ONLY TALK ABOUT THEIR AMAZING WORK BUT WHAT GOT THEM TO THIS PLACE AND WHERE DO THEY NEED TO GO AND THE BIG GOALS WE'RE AHEAD OF ALL OF US. I JOINED NCATS FOUR YEARS AGO AS WITH MANY I TOOK THE QUICK PATH. I HAD BEEN A FACULTY MEMBER FOR ABOUT 27 YEARS AND I STARTED COMPANIES BACK IN THE FIRST GOLDEN AGE IN THE '90s WHEN USED TO SAY THE MONEY WAS FREE AND IT SEEMED EASY TO START A BIO TECH COMPANY AND HAD FUN DOING THAT AND STARTED A NUMBER OF DISEASE RESEARCH AND NONPROFIT FOUNDATIONS AND PATIENT CARE FOUNDATIONS IN COMMON AND RARE DISEASES LIKE BRAIN CANCER AND HUNTINGTON'S DISEASE. WHEN I LOOK BACK AT THE VARIOUS ADVENTURES THEY'RE ALL FUN BUT IN TRYING DIFFERENT WAYS TO GET DRUGS TO PATIENTS BETTER AND FASTER. AND THERE'S LOTS OF DIFFERENT APPROACHES ONE COULD TAKE. FOUR YEARS AGO I HAD THE AMAZING OPPORTUNITY TO JOIN NCATS AND REALIZED THIS IS WHAT NCATS WAS INVENTED FOR AND THERE'S DIFFERENT WAYS TO APPROACH NEW DRUGS AND EACH ONE HAS ITS POWER AND FOCUS AND THERE WAS A BIG GAP MUCK THE 10,000 DISEASES OUT THERE AND THE MAJORITY OF WHICH ARE RARE, HOW CAN WE GET TO ALL OF THEM AND OUR EXIST IING INSTITUTIONS SET UP AND IS THE MENTION OF NCATS WAS TO PROVIDE ANOTHER KIND OF APPROACH TO KEEP THE DRUG CLASSES SLOWER THAN WE'D LIKE AND WITH LOWER SUCCESS RATE THAN WE WOULD ALL HOPE FOR. AND MOTHER NATURE IS TOUGH AND THERE'S SO MUCH WORK. THE OTHER CHALLENGES ARE OUR SOCIAL AND SYSTEM CHALLENGES. IF YOU'RE IN THE COMMERCIAL SECTOR YOU STILL HAVE A FIDUCIARY RESPONSIBILITY TO MAKE YOUR INVESTORS A PROFIT AND PUTS YOU UNDER PRESSURE AND IF YOU'RE AN ACADEMIC IT GIVES AN AMAZING FREEDOM TOE CRACK THE TOUGH NUT BUT ON THE OTHER HAND YOU HAVE TO FUND YOUR LAB AND GET GRANTS AND YOU HAVE TO PUBLISH TO STAY AROUND AND HAVE GET TENURE AND PUBLISH IN FAMOUS JOURNALS AND THIS AND THAT AND THAT GIVES A KIND OF A PRESSURE BOTH PRESSURE IN TERMS OF THE KINDS OF WORK WE CAN DO AND THE TIME LINES AND THE RISKS YOU CAN TAKE AN ALL THE COMMERCIAL PRESSURES AND ACADEMIC PRESSURES ARE NOT NECESSARILY CONSISTENT WITH THE EXTREMELY HIGH RISK AND GENERALLY SPEAKING LONG TIME LINES IT TAKES TO GET THAT INTO CLINICAL TESTING AND GET THE DRUG APPROVED. IT WAS NECESSARY TO INVENT SOMETHING LIKE NCATS AND FOUR YEARS AGO I HAD THE AMAZING OPPORTUNITY TO JOIN AND CALLED THE THERAPEUTIC DEVELOPMENT BRANCH. FRANCIS SAID IT WAS A BRIDGE AND MAYBE OVER TROUBLED WATERS BUT MOST OF US CALL IT THE VALLEY OF DEATH IN THAT SO MANY PROJECTS GET AS FAR AS THEY CAN GET IN AN ACADEMIC OR NON-PROFIT RESEARCH CENTER AND THEN COMES THE TASK OF PUTTING TOGETHER THE DATA PACKAGE FDA WILL REQUIRE BEFORE CLEARING AND IT INVOLVES RIGOROUS DATA DOCUMENTING DOES THE DRUG GET TO THE RIGHT PLACE AND DO THE RIGHT THING AND DOES IT WORK AND IS IS IT A LABORATORY MODEL AND CAN YOU MANUFACTURE IT TO BE RELEVANT FOR PATIENTS. CAN YOU PUT IT IN A FORMULATION SUCH AS A PILL AN INJECTIBLE INHALED VERSION APPROPRIATE FOR THE PATIENT'S NEED AND CAN YOU HAVE THE REGULATORY ASPECTS OF PUTTING TOGETHER SUCH A PACKAGE IN THE INVESTIGATIONAL NEW DRUG APPLICATIONS. THESE ARE HUGE THAT ARE NOT AC N -- ACCEPTABLE AND MAY BE HIGH RISK. YUL TOGETHER THERE'S SO MANY WAYS TO FAIL GOING AS FAR AS YOU CAN GET IN ACADEMIA AND TESTING IN A PATIENT AND WHY SO MANY OF US CALL THIS THE VALLEY OF DEATH AND WE LOVE LIVING IN THE VALLEY OF DEATH AND OUR GOAL IS TO PARTNER. WHETHER THEY'RE PATIENTS, ACADEMIC RESEARCHERS AND EVEN PHARMA COMPANIES OUR JOB IS TO PARTNER AND PUT TOGETHER A PROJECT TEAM TO GOOD TOGETHER AND MAKE IT THROUGH THE VALLEY OF DEATH STILL ALIVE AND KICKING ON THE FAR END. BECAUSE THIS IS OUR FIRST BIRTH DAY AND BECAUSE ON YOUR BIRTHDAY YOU GET TO BRAG A LITTLE BIT I'LL TAKE HALF A MINUTE AND SAY I THINK OUR IDEA WORKS PRETTY WELL IN THAT TO DATE WE HAVE BROUGHT OVER 40 NEW DRUG CANDIDATES INTO CLINICAL TESTING AND 40 IS A TINY NUMBER BUT ON THE BRAGGING SIDE I'D LIKE TO SAY FOR AN ENTERPRISE OF OUR SIZE AND SCALE IT'S A BIG NUMBER. WE ARE DOING SOMETHING RIGHT AND I WOULD SAY WE'RE DOING RIGHT IN THAT WE HAVE AN AMAZING TEAM AT NCATS AND WE WORK TOGETHER VERY WELL AND EMPHASIZE TEAM WORK. WE'RE NOT DESIGNED TO MAKE MONEY AND DON'T HAVE TO APPLY FOR GRANTS AND GET WE'RE DRIVEN BY DEVELOPING NEW DRUGS FOR PUBLIC GOOD FOR PATIENTS AND SO THAT GIVES US A KIND OF LIBERTY BUT IT ALSO GIVES US A KIND OF ABILITY TO SUCCEED WITHOUT THE KINDS OF DISTRACTIONS YOU MIGHT SAY THAT OFTEN MAKE THE PROCESS MORE DIFFICULT. A BIG PART IS WORKING WITH THE MOST AMAZING COLLABORATORS IN TERMS OF ACADEMIC SCIENCES AND AND PATIENT GROUPS AND PUT TOGETHER A FIRE SIDE CHAT HERE AND REFLECT MAYBE BRAG ON THINGS THAT WORK WELL BUT REFLECT ON THE PROBLEMS THAT BROUGHT US HERE AND PROBLEMS THAT REMAIN TO BE TACKLED AND HOW TO COLLECTIVELY WORK AND GET BETTER AND FASTER AT CHIPPING AWAY AT THE 10,000 DISEASES OUT THERE THAT REMAIN WITHOUT FDA APPROVED TREATMENT. THAT'S MY INTRO. OVER TO SANATH. >> I LOVE EVERYBODY AT NCATS AND THE WORK YOU ALL DO. I'M PROBABLY THE LEAST QUALIFIED PERSON TO BE TALKING ABOUT THE DRUG DEVELOPMENT BECAUSE MY BACKGROUND SIN IS IN SOFTWARE I'M THE FATHER OF A 3-YEAR-OLD BOY WHO HAS AN ULTRA RARE GENETIC MUTATION. HE IS ONE OF 10 KIDS IN THE WORLD WITH THIS DISEASE AND WHEN WE GOT OUR DIAGNOSIS TWO YEARS AGO I E-MAILED A BUNCH OF PEOPLE COLD E-MAILED AND RESEARCHERS THAT HAD ANYTHING TO DO WITH GPX4 IN THE LITERATURE AND ONE WAS AT NCATS. WE TALKED TO THEM AND A REMEMBER THE CALL AND AFTER I HUNG UP I TOLD MY WIFE I SEE HOPE THAT WE WILL EVENTUALLY GET A TREATMENT BECAUSE I HAVEN'T HAD THAT HOPE GIVEN TO ME BY ANYONE ELSE I'VE SPOKEN TO THAT EARLY IN THE PROCESS. RIGHT HERE IS AN INSTITUTION BUILT TO HELP RARE DISEASE PATIENTS LIKE US GET TO TREATMENT AND SUPPORT US IN EVERY POSSIBLE WAY WITHOUT STRINGS ATTACHED. EVERYONE HAS THEIR OWN INCENTIVE INCENTIVES AN IT ALMOST FELT LIKE THEIR INCENTIVE WAS ALIGNED WITH US IN GETTING TO US TREATMENT. SINCE THEN WE'VE WORKED WITH THEM ON A VARIETY OF PROJECTS FROM UNDERSTANDING GPX4 BIOLOGY AND DOING HIGH THROUGHPUT SCREENING AND FELT LIKE THE DOOR WAS ALWAYS OPEN. THAT'S POWERFUL BECAUSE THE PATTERN WE TAKE AS PATIENT LEADERS IT'S A JOURNEY NO ONE TAUGHT US TO TAKE WE ARE NOT QUALIFIED. WE DON'T GO TO SCHOOL. WE ARE NOT GIVEN OFFICIAL TITLES. THERE'S NO CAREER LADDERS HERE. IT'S A JOURNEY WE GOT INTO BY NOT BY CHOICE BUT BY LOOK. WE NEED PARTNERS THAT WORK WITH US AND I'M HAPPY I CAN WORK WITH NCATSES AND I KNOW A LOT OF OTHER PATIENT DO AS WELL. MY BACKGROUND STARTED WITH ONE THE NCATS GROUP AND SINCE THEN WE STARTED A FOUNDATION AND RAISED A LOT OF MONEY AND INVESTED IN HIGH THREE-POINT DRUG SCREEN. ONE OF THE FIRST PROJECTS WE DID WAS TO DEEP DIVE IN THE LITERATURE TO IDENTIFY DRUGS THAT COULD BE POTENTIALLY BENEFICIAL FOR MY SON'S POSITION. WE GOT MY SON STARTED ON A FEW SUPPLEMENTS WEEKS AFTER THE DIAGNOSIS AND ADDED MORE TO THE LIST. SO FAR HE'S BEEN ON A COCKTAIL OF FIVE DIFFERENT DRUGS. NEXT WEEK WE'RE STARTING OUR SIXTH DRUG AND HOPE IT WILL BE THE MAGIC IF NOT WE'LL CONTINUE DOWN THE PATH OF INVESTIGATING OTHER SMALL MOLECULE DRUGS TO REPURPOSE TO POTENTIALLY HAVE BENEFITS FOR MY SON. IN PARALLEL WE'RE WORKING ON GENE REPLACEMENT THERAPIES AN INVESTIGATING THE BASIC SCIENCE TO CRACK THAT TOUGH NUT AND HOPE TO HAVE A CANDIDATE ONE DAY THAT WILL GIVE PATIENTS LIKE MY SON A CHANCE AT A MEANINGFUL QUALITY OF LIFE BECAUSE CURRENTLY HE'S SUFFERING FROM A BUNCH OF MEDICAL ISSUES AND MORE IMPORTANTLY HE DOESN'T WORK AND LIVE LIKE A 3-YEAR-OLD. HE CANNOT SIT ON HIS OWN OR HOLD OBJECTS EN HIS HAND AND NOT CAN WALK OR TALK OR EAT ANYTHING BY MOUTH. THE KIND OF QUALITY OF LIFE YOU'D EXPECT FROM A 3-YEAR-OLD IS NOT REMOTELY WHAT MY SON HAS TODAY. MY MISSION HAS BEEN ALWAYS TO FIND A TREATMENT FOR MY SON AND IN THAT PROCESS I'VE COME ACROSS A LOT OF INEFFICIENCIES IN THE ECO SYSTEM I'M HOPING TO SOLVE WITH THE OPEN TREATMENT FOUNDATION A NEW NON-PROFIT ORGANIZATION I STARTED WITH OTHER RARE DISEASE FINDERS IN THE SPACE AND USE SOFTWARE TO SOLVE THE INEFFICIENCIES AT A LARGER SCALE AND ONE OF THE INITIATIVES STARTED IN MARCH WAS TO MAKE IT EASIER TO BUILD GENE REPLACEMENT THERAPIES AND START EIGHTED PILOT PROGRAM WITH FOUR DIFFERENT FOUNDATIONS AND NCATS IN COLLABORATION HAS A COMPANION PROGRAM AROUND BUILDING GENE REPLACEMENT THERAPIES AND WE'RE STARTING ANOTHER INITIATIVE TO START COUNTING THE NUMBER OF PATIENTS EACH DISEASE HAVE AND EACH FOUNDATION THAT HAS A ROUGH ESTIMATE OF THE NUMBER OF PATIENTS THEY HAVE, WOULD LOVE TO MULTIPLY THAT NUMBER BY 10 AND THEY ALWAYS FEEL LIKE THERE'S MORE PATIENTS OUT THERE BUT WE DON'T HAVE AN RACK RAT NUMBER. ANOTHER INITIATIVE I'M STARTING TO WORK WITH IS TO GET A COUNT AND START COUNTING THE NUMBER OF PATIENTS WE HAVE IN ALL RARE DISEASES. WE NOT ONLY HAVE AN ESTIMATE BUT PREVALENT COUNTS PUBLICLY AVAILABLE ANYONE CAN VIEW AND ACCESS AND TO FORM THE BEDROOM OF FUTURE RARE DISEASE DRUG DEVELOPMENT. THAT'S WHAT I'M PERSONALLY TRYING TO DO AND FOLKS LIKE NCATS HAVE BEEN AN INCREDIBLE PART AND THIS IS WHERE IT'S DIFFERENT AND UNIQUE IN THAT SENSE. >> THANK YOU, YOUR CLEARLY NOT THE LEAST QUALIFIED BY HUGE MEASURES AND WE HAVE PHRASE HOW IMPORTANT TEAM PLAY SAND TASE TRUE AND WHAT YOU'RE DOING REFLECT THE CHALLENGE WE ALL HAVE THAT COLLECTIVELY ALL THE RARE DISEASES TOGETHER CAN'T BE REINVENTING EVERY TOOL ALL OVER AGAIN EACH TIME. SO IT'S SO EXCITING THAT WE'RE WORKING TOGETHER AND LOOK FORWARD TO MANY MORE THINGS TO TACKLE IN THE FUTURE. >> FIRST, HAPPY BIRTHDAY NCATS IT'S A PLEASURE TO BE HERE TO CELEBRATE THIS IMPORTANT ANNIVERSARY. THANK YOU FOR THE CHANCE TO PARTICIPATE. SO WHO AM I AND WHAT AM I DOING HERE? I'M A NEUROLOGIST AND GENETICIST BY TRAINING. IT'S MY PLEASURE TO LEAD A GROUP AT BOSTON'S CHILDREN HOSPITAL. IN THE PAST OUR GROUP HAD FOCUSSED FOR A LONG TIME ON USING THE TOOLS OF HUMAN GENETIC TO DISCOVER FUNDAMENTAL CAUSES OF HUMAN DISEASES ESPECIALLY THOSE AFFECTING KIDS. THERE'S VAST NUMBERS OF RARE DISEASES THAT HAVE VERY LOW SMALL NUMBERS OF INDIVIDUALS AFFECTED AND MANY RARE DISEASE FOUNDATIONS WOULD LOVE TO MULTITHE NUMBER OF RARE DISEASES BY 10 TO ATTRACT BIO TECH ATTENTION. IT'S A FUNDAMENTAL PROBLEM WE'VE BEEN CAPTIVATED BY WHAT IF WE DIDN'T HAVE TO DO THAT? AND WHERE WE CAN MOTIVATE AND DEPLOY MEDICINES FOR PATIENTS WITHOUT NEETH TO HAVE A -- FEEDING A THOUSAND PATIENTS TO ATTRACT A BIO TECH COMPANY? OUR THINKING BEGAN WITH A STORY WE GOT INTO THIS WORK WHEN WE MET A YOUNG CHILD MILA AND DIAGNOSED WITH A RARE AND DEGENERATIVE DISEASE FOR WHICH THERE WERE NO TREATMENTS. SHE HAD A RARE SUB TYPE AND WITH TWO DOZEN PATIENTS IN THE WORLD KNOWN TO HAVE THAT THERE WAS NOT A THERAPY. COLLABORATING WITH HER TEAM AT THE UNIVERSITY OF COLORADO AND OTHERS WE WERE ABLE TO USE WHOLE GENOME SEQUENCING TO IDENTIFY A SPECIFIC MUTATION IN HER AND HER SPECIFIC MUTATION NOT ONLY SOLVED HER CASE AND PROVED HER DIAGNOSIS BUT UNLOCKED AN UNUSUAL OPPORTUNITY THE OPPORTUNITY THAT DRIVES OUR WORK TODAY. THAT OPPORTUNITY IS A RECOGNITION THAT CERTAIN CLASSES OF MUTATIONS ARE AMENABLE TO INTERVENTION WITH A NEW GENERATION OF GENETICALLY TARGETABLE THERAPIES. AND THE THERAPIES WITH GENOME EDITING AND OTHERS ARE PLATFORM TECHNOLOGIES THAT CAN BE USED TO CORRECT DEFECTS ALMOST IRRESPECTIVE OF THE DISEASE AND GENE IN WHICH IT LIES. AND THAT TURNED OVER A YEARLONG RACE INTO INDIVIDUALIZED TREATMENT FOR A HER A YEAR AFTER WE MET HER AND THAT THERAPY AS WE REPORTED IN THE SCIENTIFIC LITERATURE RESULTED IN SIGNIFICANT IMPROVEMENTS IN HER QUALITY OF LIFE AND REDUCTION OF UNDERLYING SESURES SHE WAS SUFFERING FROM AT GREAT FREQUENCY AND -- SEIZURES AT GREAT FREQUENCY AND HELPED HER DURING A CRITICAL TIME IN HER DISEASE WHEN THINGS WERE ONLY DESTINED TO GO DOWN HILL. SHE IS OUR AVATAR AND EXAMPLE OF THE TYPE OF PROMISE TECHNOLOGY COMBINED WITH COLLABORATION THROUGH NCATS AND OTHER MEMBERS OF THE ECO SYSTEM CAN BRING AND OUR LAB IS FOCUSSED ON FINDING MORE FARM HAS AND NOW LAUNCHED HALF A DOZEN PROGRAMS TO TREAT PATIENTS WITH INDIVIDUALIZED THERAPIES BASED OFF THIS TECHNOLOGY AND WE'RE FORTUNATE TO COLLABORATE WITH OTHERS AND FDA FOUNDATIONS IN THE UNITED STATES AND UNITED STATES AND ACADEMICS AND HOSPITALS AND FAMILIES AND PATIENTS TO TRY TO FORTIFY THIS TYPE OF OPPORTUNITY TO SEE IF WE CAN DEVELOP PROCESSES FOR INTERVENING TO CHALLENGE OUR EXISTING PARADIGMS FOR HOW DRUGS ARE APPROVED BUT MAY MODERNIZE THE PROCESS AT THE SAME TIME. I'LL LEAVE MY INTRODUCTORY REMARKS WITH THAT. THANK YOU AGAIN FOR THE CHANCE TO BE HERE AND CHAT WITH YOU ALL. >> THANK YOU. YOU'RE A KIND AND MODERN TREATMENT AND MILEA WAS ONLY ONE YEAR, THAT IS FAST AND OF YOU NAY KNOW THE AVERAGE TIME IT TAKES -- MAY KNOW THE AVERAGE TIME IT TAKES FROM DISCOVERY TO APPROVAL IS 14 YEARS AND A COUPLE BILLION DOLLARS AND THAT NEEDLE HASN'T MOVED. IF YOU LOOK BACK TO THE LEFT DECADE 20, 30, 40 YEARS IT'S ALWAYS BEEN 14 YEARS AND THE STANDARD DEVIATION IS VERY SMALL. THE WORK IN FDA CLEARANCE FOR A NEW DRUG IN ONE YEAR IS ASTONISHING. ONE OF THE MOST AMAZING ACHIEVEMENTS IN TRANSLATIONAL MEDICINE I'M AWARE OF IN THE MANY YEARS. KUDOS AND THE REASON WE'RE SO EXCITED TO COLLABORATE AND AGGREGATE MORE AND MORE PEOPLE IS WHAT YOU'RE SAYING, HOW CAN WE MAKE THIS WORK FOR MANY MORE PATIENTS, HUNDREDS OR THOUSANDS MORE AND THOUGH IT HAS BEEN A REAL GOAL TO CONSTRUCT COMMERCIAL STRATEGIES WHERE THE COMMERCIAL SECTOR WOULD BE COPACETIC IN THE BIO TECH SIDE TO STUDY MORE AND MORE RARE DISEASES IT'S A LONG TAIL OF RARITY. AT SOME POINT IT'S JUST NOT GOING TO BE THE ECONOMICALLY FEASIBLE AT ALL AND WE AS A COMMUNITY OF PATIENTS, SCIENTISTS, PHYSICIANS, ETCETERA, NEED TO BAND TOGETHER TO FIGURE OUT HOW TO ADDRESS THIS WITH OTHER STRATEGIES. IT WILL BE AN EXCITING TIME IN WORKING TOGETHER TO DEVELOP PLATFORMS THAT ARE OBVIOUSLY PLATFORMS AND THEN OTHER DRUG STRATEGIES THAT MAY NOT OBVIOUSLY BE A PLATFORM OR MAY NOT BE A PARADIGM AND ONE THING WE'RE DOING IS FINDING COLLABORATIVE LIKE YOU GUYS AND SHOWED IF YOU ADDRESS ONE DISEASE WITH THE STRONG SCIENTIFIC HYPOTHESIS AND PERHAPS AN IMPROVED OR RISK ER, FASTER APPROACH COULD YOU SOLVE ONE DISEASE AND GET A TREMENT FOR ONE DISEASE OTHERS CAN LEARN FROM AND A THEME THAT'S COME UP SPOKEN ABOUT AND NCATS IS TRYING TO DO MORE OF IS FIND BETTER WAYS TO DO OPEN SCIENCE PARTICULARLY IN RARE DISEASES WHERE THERE ISN'T A COMMERCIAL PROPOSITION IN THE FUTURE. WE HAVE TO BE MORE AND MORE PB AND NOT LET HISTORICAL CONVENTIONS KEEP US FROM SHARING DATA. >> FUNDAMENTALLY THE EFFORTS YOU'VE LAUNCHED IS SUCH A PERFECT ENCOLLAPSELATION OF THAT. THE PROCESS WE'RE TALKING ABOUT IS A NEED TO TAKE RISK IN HOW DRUGS ARE DEVELOPED. IT'S A BUSINESS MODEL RISK. A GIVEN MOLECULE IS ADVANCED THROUGH PAINSTAKING BASIC SCIENCE AND EXTENSIVE PRE-CLINICAL STUDIES AND LARGE CLINICAL TRIALS AND THAT THAT EFFORT IS POURED IN THE MOLECULE ITSELF AND REPAY THE GREAT INVESTMENT IT TOOK TO GET IT TO THAT POINT IN THE FIRST PLACE. AND NOW WE HAVE THE OPPORTUNITY TO BASE THIS OFF PROCESS WHERE IT'S NOT SO MUCH THE MOLECULE ITSELF BUT THE PLATFORM IN WHICH THE MOLECULE WAS BUILT. IN THAT PARADIGM SHARING DATA IS NOT ONLY NATURAL BUT REQUIRED IN ORDER FOR THE PLATFORM TO GROW. UNDERSTANDING THAT REQUIRES TAKING BUSINESS RISK AND WHY NCATS AND FOUND S LIKE OPEN TREATMENTS ARE IN SUCH A PERFECT POSITION TO TAKE THE CREATIVE RISKS TO THINK ABOUT BUSINESS A LITTLE BIT DIFFERENTLY AND ADVANCE THAT IDEA IN ACADEMIC MEDICAL CENTERS AND HOPEFULLY INVESTIGATORS LIKE MYSELF. >> MY HOPE IS FOR PROFIT BUSINESSES WOULD ACCEPT THAT RISK AND BRING IN THE CAPITAL MONEY INTO IT BECAUSE YOU SEE THE BIG COMPANIES OF THE WORLD THAT ARE TRILLION DOLLAR COMPANY LIKE APPLE HAVE PRODUCTS IN ALMOST EVERY PERSON'S HAND AND WHY CAN'T THAT BE THE NEXT RARE DISEASE COMPANY? THE FUNDAMENTAL PROBLEM IS THE BUSINESS MODEL RISK AND HOPEFULLY AND EVENTUALLY WE COULD PROVE TO THE ENTITIES THIS IS A VALID RISK TO TAKE AND THERE'S A PAYOFF AT THEN OF IT AND HAVE MORE BIO TECH COMPANIES THAN WE HAVE TODAY AND WHAT WOULD THAT LOOK LIKE FOR PATIENTS. >> THERE'S MANY WAYS TO WORK TOGETHER AND CERTAINLY WE'RE PROUD TO SERVE AS A CONVENING POINT FOR THE IDEAS AND WOK TOGETHER AND GET HELP TO WORK TOGETHER AND MOVE IT ON THE NEXT STAGE. I THINK WE'RE WITHIN A MINUTE OR TWO OF HAVING TO CLOSE OUR FIRE SIDE CHAT. ANY CLOSING COMMENTS? >> THERE'S A WHOLE JOURNEY OF FINDING TREATMENT FOR RARE DISEASE PATIENTS MAY LOOK INSURMOUNTABLE BUT I'VE SEEN HOPE AND A LOT OF DRAMATIC CHANGE IN THE ECO SYSTEM. HOPEFULLY AT THE 15th ANNIVERSARY MEETING THE LEVEL OF HOPE WILL BE DIFFERENT KEEPING FINGERS CROSSED I THINK WE'LL GET THERE. >> THAT'S A WONDERFUL THOUGHT. THANK YOU VERY MUCH. WE'VE BEEN WORKING IN THIS RARE DISEASE SPACE AS A SILENT EPIDEMIC BUT THE ONGOING PANDEMIC IS SHOWING THE WORLD WAYS PLATFORM SCIENCE CAN CHANGE THE WAY WE DEVELOP TREATMENTS AND VACCINES WORKING WITH VARIANTS OF THE COVID VIRUS AND ACCELERATING OUR ABILITY TO RESPOND TO A SERIES OF GENETIC CHANGES. I THINK THE IDEAS WE'RE SEEING A COALESCENCE OF THE RARE AND GENERATIONALLY DEFINING PANDEMIC THAT I THINK WILL REINFORCE THE SAME THING WE'RE TALKING ABOUT AND I'M OPTIMISTIC AT THE 15th OR 20th YEAR ANNIVERSARY WE'LL BE ABLE TO REPORT GOOD PROGRESS. GOOD THANK YOU. I AGREE WE'RE COMING TO THE CONVERGENCE AN ALIGNMENT OF THE CHALLENGES FACING AN ULTRA RARE DISEASE AND HUGE DISEASE FOR WHERE THE CLOCK IS TICKING SO LOUDLY AND MANY CHALLENGES ARE THE SAME AND IT WOULD BE WONDERFUL TO HAVE A MEETING OF THE CHALLENGES AND WAYS TO OVERCOME THE CHALLENGES. THANK YOU AGAIN. I PERSONALLY ENJOYED OUR FIRE SIDE CHAT. THANK YOU FOR TUNING IN. IT'S TIME TO PASS ON TO THE NEXT SPEAKER, MARC, WHO CAN TELL US ABOUT THE AMAZING PROJECTS AT NCATS DESIGNED TO SUPPORT THE DEVELOPMENT OF NEW CANDIDATES FOR A COMMON DISEASES AND URGENT DISEASES AND ULTRA RARE DISEASES. MARC. >> THANK YOU SO MUCH, DON. WELCOME TO THE NCATS CELEBRATION. I'M MARC FERRER AND HEAD THE LAB AT THE INTRAMURAL LABS AND SPENT MOST MY CAREER DEVELOPING BIOLOGICAL ASSAYS FOR DEVELOPMENT AND I CAME TO NCATS FOR THE VERY INSPIRING STORIES HOW THE PATIENT GROUPS AND NCATS INVESTIGATORS ARE COMING TOGETHER AND FORMING COLLABORATIVE PROJECTS TO MAKE AN IMPACT IN THE PUBLIC HEALTH AND TREATING DISEASES. THE SECOND ASPECT THAT RESONATED WITH ME WITHIN NCATS IS LOOKING AT THE DRUG DISCOVERY PROCESS AS A PROBLEM THAT NEDZ TO BE INVOLVED. BLENDING CHEMICAL AND ENGINEERING AND INFORMATIC APPROACHES TO SOLVE THE PROBLEM THAT IS APPEALING TO ME. WE TALKED ABOUT INNOVATION AT NCATS. WE HAVE A LOT OF DISCUSSIONS ABOUT WHAT IT MEANS TO DO INNOVATION IN DRUG DISCOVERY. TO ME SCIENTIFIC INNOVATION IS A PROCESS OF IDENTIFYING THE PROBLEM WE'RE TRYING TO SOLVE. THEN COMING UP WITH IDEAS AND NEW TECHNOLOGIES AND NEW APPLICATIONS WE CAN USE TO SOLVE THE PROBLEM. THE LAST PART IS IMPLEMENTATION AND HAVING IDEAS WITHOUT IMPLEMENTATION WON'T SOLVE THE PROBLEM AND WHAT I'LL TELL YOU IS ABOUT AN EXAMPLE OF HOW WE WENT ABOUT ESTABLISHING A PLATFORM TO TRY TO IMPROVE THE WAY WE TEST COMPOUNDS AND DEVELOPING ASSAY PLATFORMS RELEVANT AND HOPEFULLY WILL HELP US IMPROVE THE PROCESS OF DRUG DISCOVERY. STARTING WITH A PROBLEM IS THE DRUG DISCOVERY PROCESS AND NOT VERY SUCCESSFUL AND IT HAS A VERY HIGH FAILURE RATE. 10% OF THE DRUGS ENTER CLINICAL TRIALS BECOME DRUGS. THIS IS BECAUSE EITHER THEY HAVE NO EFFICACY OR HAVE A TOXIC EFFECT. WE NEED TO TRY TO UNDERSTAND WHY DO WE HAVE THIS HIGH FAILURE RATES AND THERE'S REASONS BUT PART OF THE PROBLEM IS THAT WHEN THIS EXPANDED TO CLINICAL TRIALS THERE'S BEEN TESTED THROUGH ASSAYS AND ANIMAL MODEL AND THEY WORKED. THE PROBLEM SEEMED TO BE THE ASSAYS WE CURRENTLY USE ARE KNOW PREDICT I OF WHAT A DRUG WILL DO AND THE QUESTION IS HOW DO WE INCREASE THE PREDICTABILITY OF THESE ASSAYS. WE USE CELLS THAT HAVE BEEN GROUND AND PASSED FOR YEARS. THE CELLS ARE GROWN ON PLASTIC BECAUSE IT'S EASY TO MAKE LARGE AMOUNTS OF CELLS TO USE FOR WHAT YOU HEARD LIKE HIGH THROUGHPUT SCREENING WHICH TESTS COMPOUNDS TO TEST IF SOME ARE ACTIVE IN A PARTICULAR ASSAY AND BY GROWING THE CELLS IN THE PLASTIC ENVIRONMENT AND MONOLAYERS IT'S NOT WHAT'S IN YOUR BODY. THE CELLS IN YOUR BODY ARE GROWING IN SCAFFOLDS AND THE CELLS IN YOUR TISSUES AND ORGANS FUNCTION BY INTERACTING WITH OTHER CELL TYPES AND SOMETIMES IT'S IN THE SAME TISSUE OR OTHER TISSUES AND ORGANS AND THERE'S LONG DISTANCE COMMUNICATION BETWEEN CELLS AT THE END OF THE DAY CELLS IN YOUR BODY HAVE DIFFERENT BIOLOGY AND BECAUSE THEY HAVE DIFFERENT BIOLOGY THEY RESPOND DIFFERENT TO DRUGS. AND WE NEED TO DEVELOP ASSAY ARE MORE WHAT WE CALL PHYSIOLOGICALLY RELEVANT AND DEVELOP ASSAYS IN WHICH THE CELLS ARE MORE IN AN ENVIRONMENT THAT MIMICS THE REAL TISSUE IN YOUR BODY. THIS IS WHAT OF THE CELLULAR MODELS AND AS THE DIFFERENT FLAVORS OF MODELS AND HOW WE GOT INTERESTED IN THE TISSUE IIO -- TISSUE BIO PRINTING. IT'S SIMILAR TO THE PRINTER AT HOME AND YOU HAVE CARTRIDGES WIN INK AND A COMPUTER YOU USE TO CREATE A DESIGN DRAWING AND THEN THIS DESIGN IS SENT TO THE PRINTER AND THE PRINTER USES THE CARTRIDGES. WHEN WE DO TISSUE BIO PRINTING IT'S SIMILAR IN THE SENSE WE HAVE CART RIDGES AND THE CELLS ARE MIXED WITH SOME HYDRO GEL THAT WE CALL BIO INK AND USE SOFTWARE TO CREATE PATTERNS OF TISSUES LIKE THE NATIVE TISSUES. WHAT WE'RE NATIONALLY ABLE TO DO IS PUT THEM IN THE HYDRO GELS AND CREATE THE BIOPRINTED TISSUES WE HOPE WILL LOOK LOOK AND FUNCTION LIKE -- LOOK LIKE AND FUNCTION LIKE THE TISSUES IN YOUR BODY. IT IS MORE COMPLICATED THAN THAT BUT IN A NUTSHELL THAT'S WHAT WE'RE DOING AND THE POTENTIAL AND REALITY OF THE TECHNOLOGY WE THINK IS USEFUL LETS YOU TAKE CELLS THAT IN MANY CASES WE USE CELLS DEVISED DIRECTLY FROM PATIENTS AND WE HAVE CELLS DIFFERENTIATED FROM STEM CELLS BUT TRYING TO CREATE TISSUES WITH CELLS CLOSE TO THE PATIENT'S. AND FIRST WE LEARN HOW TO CREATE A TISSUE WE CALL NORMAL AND THAT WOULD BE ANY RELEVANT TISSUE THAT'S IMPORTANT FOR YOUR DISEASE AND COME IN WITH SOME SORT OF CELL TYPE FROM A PATIENT WHETHER RARE DISEASE OR CANCER CELL AND CREATE THE DISEASE MODELS AND TAKE THE TISSUES AND COME IN WITH DRUGS AND ANTIBODIES AND OTHER THERAPEUTICS AND TRY TO CREATE THE PHENOTYPE IN THE CONTEXT OF THE TISSUE LIKE STRUCTURE. ONE EXAMPLE IS WE TRIED TO WORK AND A TISSUE MODEL TO LOOK AT LUNG CANCER AND CANCER METASTASIS IN THE LUNG AND STARTED CREATING LUNG TSHZ AND THE COVID-19 EPIDEMIC STARTED. BY CREATING FIRST THE NORMAL TISSUE WE WERE ABLE TO CREATE THE LONG TISSUES FROM CANCER TO STUDYING THE INFECTIVITY IN THE LUNG TISSUE MODELS. IT'S BEEN A VERY INTERESTING JOURNEY LOOKING AT DIFFERENT VARIANTS. CAN WE SEE INFECTIVITY AND PRODUCE THE DIFFERENCES IN INFECTIVITY IN THE VARIANTS OF TE TISSUES AND WE'VE SEEN GOOD CORRELATION BETWEEN ENEFFECTIVITY -- INFECTIVITY IN THE VIRUS OF THE TISSUES. THERE'S A CLINICAL COOL IN VITRO CORRELATION. WE ALSO USE THESE TISSUES AND YOU PROBABLY HEARD SOME OF THE COVID-19 PATIENT HAVE CYTOKINE STORM AND STRONG IMMUNE RESPONSE TO THE VIRUS. WE CAN LOOK AT CYTOKINES AND CHEMOKINES AND WERE ABLE TO DETECT SECRETION OF THESE UPON INFECTION OF THE TISSUES VERY MUCH MIMICKING WHAT HAS BEEN SEEN IN CLINIC. LASTLY, CAN WE USE THESE MODELS TO SCREEN FOR DRUGS THAT HAVE ANTIVIRAL ACTIVITY? WE WERE ABLE TO TEST COMPOUNDS LIKE REMDESIVIR BUT OTHER DRUGS THAT WERE VERY MUCH IN CONSIDERATION OF TREATMENT AND WHEN TESTED IN THIS MODEL IT WAS CLEAR THESE DRUGS WOULD NOT BE EFFICACIO EFFICACIOUS. THE MODELS WERE PREDICTIVE THE CLINICAL MANIFESTATIONS IN FECT INFECTIVITY AND WE'RE EXTENDING TO OTHER MODELS BECAUSE THE APPROACH IS VERSATILE AND LED US TO CREATE DIFFERENT TISSUES AND DIFFERENT MANIFESTATIONS IN MATERIALS OF BLOOD VESSELS AND VASCULARIZED FEATURES WING TISSUES OF DIFFERENT PHYSIOLOGICAL COMPLEXITY. WE CREATE THE NORMAL TISSUES AND TRIED TO VALIDATE THEM AND BENCHMARK THEM WITH CLINICAL SAMPLES. IT'S A LITTLE BIT OF AN UNGLAMOROUS WORLD BUT WHERE NCATS CAN TAKE A DIFFERENCE LIKE SOMEONE MENTIONED THIS MORNING IT'S IMPORTANT TO DO PUBLICATION BUT THERE'S A LOT OF WORK TO MAKE THESE THESE TISSUE MODELS ROBUST AND SCALABLE AND REPRODUCIBLE ENOUGH TO USE THEM FOR A DRUG DISCOVERY PIPELINE. WE'VE HAD WORK TO ESTABLISH THE PREDICTABILITY WHAT PEOPLE CALL THE PREDICTABILITY OF THE MODELS. CAN WE SAY THE MODELS WILL CREATE DRUG RESPONSES IN CLINIC AND ONE ASPECT WE'RE INTERESTED IN IS PERSONALIZED MODELS AND WILL ENABLE THE TISSUE TO CREATE THE CELLS OF ONE PATIENT WHETHER IT'S RARE DISEASE OR CANCER PATIENT AND INVESTIGATE THE DRUG RESPONSES IN THE PRECISION MEDICINE TYPE OF MODELS AND AT THE END CAN WE USE THIS TO GUIDE HOW TO DO A CLINICAL TRIAL AND BE BETTER AT HOW WE PUT DRUGS IN CLINICAL. IT IS BOTH TE ROBUSTNESS AND KALE SCALE UP OF THE MODELS. IT'S THE CLINICAL BENCHMARKING AND ASSESSING THE MODELS AND WILL LEARN HOW PREDICTIVE THEY ARE AND WHETHER IT WILL HELP THE USE OF ANIMAL MODELS AND HOPEFULLY WE'LL BE ABLE TO USE ANIMAL MODELS IN THE FUTURE. THAT'S THE VISION. AND TALK ABOUT THE OTHER TECHNOLOGIES TO CREATE 3-D CELLULAR MODELS. DAN. >> THANK YOU, MARC AND HAPPY TO BE HEAR AND HAPPY TO CELEBRATE THE OCCASION. I'M DAN TANGLE AND HAPPY TO TELL YOU ABOUT THE PROGRAM WHICH IS A PROGRAM FRANCIS COLLINS WE DETERMINED WOULD BE A GOOD FIT AT NCATS WHEN NCATS WAS INITIALLY ESTABLISHED. A AND WE'LL LOOK AT 28% OF THEM FAIL BECAUSE OF SAFETY AND TOXICITY PROFILES. AND THAT HAS LED TO A LONG DEVELOPMENT TIME LINE OF 10 TO 15 YEARS TO BRING A DRUG TO MARKET AND $2.6 BILLION IN COST. AND THE MAIN CULPRIT IN THAT CAN BE ATTRIBUTED TO THE ANIMAL MODELS WE'VE BEEN USING IN DRUG DEVELOPMENT AND THE GRAPH INDICATES A POOR CONCORDANCE BETWEEN ANIMAL MODELS AND HUMAN MODEL A NUMBER OF RARE DISEASES AND IN THIS CASE AN EXAMPLE WOULD BE LIKE BART SYNDROME AND FORMATIONS AS WELL AS COMMON DISEASES SUCH AS ALZHEIMER'S, PARKINSON'S AND EVEN COMPLEX DISEASES LIKE DIABETES THAT CONFIDENT OF MULTIPLE ORGAN PATHOLOGY AND WE HAVE ALSO BEEN FORTUNATE TO RESPOND TO NATIONAL HEALTH EMERGENCIES. AND TISSUE CHIPS FOR OVERDOSE AND ADDICTION AND NOCICEPTION AND RECENTLY WITH THE RECENT HEALTH CRISIS WITH THE PANDEMIC TISSUE CHIPS TO MODEL INFECTION TO LOOK AT HOW VARIOUS ORGAN SYSTEMS AND PATHOLOGIES ARE BE AFFECTED AND TESTING FOR ANTIVIRAL AGENTS AND VACCINE. IN ADDITION WE HAVE USED TISSUE CHIPS IN COLLABORATION WITH NASA AND THE ADVANCEMENT OF SCIENCE AND SPACE TO DEVELOP MODELS TO INFORM ACCELERATING AGING USING THIS ENVIRONMENT OF MICROGRAVITY IN THE INTERNATIONAL SPACE STATION AND LAST YEAR THERE WAS A NEW PROGRAM FOR CLINICAL DESIGN AND PRECISION MEDICINE AND THE TISSUE CHIPS ARE BEING USED TO BE POPULATED FROM RARE DISEASES OR COMMON DISEASES AND MUTATION SPECTRUM AND THE DEMOGRAPHIC OF PATIENT POPULATION AND USING THAT FOR INFORMING HOW CRITERIA CAN BE DESIGNED AND TO BE ABLE TO DEVELOP A CLINICALLY RELEVANT BIOMARKER TO LEAD THEM TO PERSONALIZED MEDICINE APPROACH. NCATS IS ALSO COGNIZANT OF DEVELOPING TOOLS AND ADOPTION BY THE COMMUNITY AND IMPACTING DRUG DEVELOPMENT. IN THIS PARTICULAR PROGRAM OF TISSUE CHIPS WE HAVE LOOKED AT TECHNOLOGY ADOPTION IN THE 10 YEARS OF THE PROGRAM THAT EXISTED WE HAVE MADE QUITE A BIT OF HEADWAY IN TERMS OF ADOPTING THE TECHNOLOGY TO WIDESPREAD USE. THE KEY ENABLERS FOR THE VAST ADOPTION AND COMMERCIALIZATION IS THE NEW TECHNOLOGY THE TISSUE CHIPS SHOULD MEET THE MARKET NEEDS. IT NEEDS TO HAVE A PARTICULAR CONTEXT OF USE BY DEFINITION BY THE FDA WE NEED ENGAGEMENT WITH END USERS SINCE AND PHARMACEUTICAL AND DRUG DEVELOPMENT COMPANIES AND FOR GENERAL USE THERE HAS TO BEES OF USE AND COST EFFICIENCY. IN THIS REGARD WE HAVE PARTNERED WITH A CONSORTIUM AND I.Q. STANDS FOR INNOVATION EQUALITY. IT'S A CONSORTIUM OF RIGHT NOW ABOUT 21 PHARMACEUTICAL COMPANIES INTERESTED IN THE USE OF TISSUE CHIPS FOR SYSTEM TECHNOLOGY AND I'LL TELL YOU MORE HOW THEY'RE USING THAT IN THE NEXT SLIDE. WE'VE ALSO INDICATED WORKED FROM THE VERY BEGINNING OF THE PROGRAM WITH THE FDA IN TERMS OF HOW THIS TECHNOLOGY CAN BE QUALIFIED AS DRUG DEVELOPMENT TOOLS AND BE INFORMED IN TERMS OF HOW THEY DEVELOP AND HOW COULD BE POSITIONED IN TERMS OF THE THREE Rs FOR ANIMAL USE, REPLACEMENT, REDUCTION AND REFINEMENT. MOREOVER, AS I'VE MENTIONED IN THE PREVIOUS SLIDE WE WORKED WITH OTHER PARTNERS LIKE NASA AND NOT ONLY MODELLING AGING BUT IN TERMS OF TAKING THE KNOWLEDGE BASE OF SPACE ENGINEERS AND PLATFORM AND PAY LOAD DEVELOPERS IN TERMS OF HOW TO MINIATURIZE AND AUTOMATE SYSTEMS THAT CAN BE TAKEN INTO SPACE AND SO WE CAN TRANSLATE THOSE KNOWLEDGE IN TERMS OF USING THE ATTORNEY GENERALS BETTER ON EARTH AND ONE THING NCATS IS INTERESTED IN IS MAKING THE INFORMATION PUBLICLY AVAILABLE SO WE CREATED THE DATABASE ARE IN ORDER TO LOOK AT THE ROBUSTNESS AND REPRODUCIBILITY OF THE SYSTEMS WE HAVE CENTERS ONE INITIALLY HOUSED AT M.I.T. AND THE SECOND AT TEXAS A&M AND DEMONSTRATES HOW REPRODUCIBLE THEY AND MAKES THE SYSTEM WIDELY AVAILABLE. THE PLATFORMS ARE MODELS THAT ONCE SUPPORTED BY NCATS WE TRANSLATED TO SELF-SUSTAINING BUSINESS MODELS AND LASTLY WE ALSO ESTABLISHED A MICROPHYSIOLOGICAL SYSTEM WORLD SUMMIT THE FIRST CONFERENCE TO BE HELD IN JUNE OF NEXT YEAR. IT WILL BRING TOGETHER TISSUE DEVELOPERS AND WHERE THE ACTIVITY HAS GROWN IN EUROPE AND ASIA AND AUSTRALIA. I MENTIONED PHARMA HAVE BEEN TAKING AN ACTIVE INTEREST AND HAVE BEEN USING TISSUE CHIPS IN TERMS OF DRUG DEVELOPMENT IN TERMS OF TARGET IDENTIFICATION AND PRE-CLINICAL SAFETY AND EFFICACY AND IN PHARMACO KINETICS AND INTOXICOKINETICS. AND LASTLY WE HAVE THROUGH THE SMALL BUSINESS ACTIVITIES ENCOURAGED THE FORMATION OF SPIN OFFS AND START OF COMPANIES AND THIS SHOWS THERE'S 20 OR SO COMPANIES CENTERED ON ORGANOIDS AND COMMERCIALIZING THEM IN TERMS OF CRO TYPE SERVICE OR CONSUMABLES. AND TO SHOW YOU WHERE TISSUE CHIPS CAN BE POSITIONED IT CAN GO WITH IDENTIFICATION AND ALL THE WAY TO CLINICAL STUDY. AND RECENT STUDIES HAVE SHOWN BY 2024 DRUG DEVELOPMENT COSTS WOULD RISE TO $213 BILLION AND THERE'S BEEN STUDIES THAT SHOWS IF TISSUE CHIPS WHICH ARE BEING DEPLOYED IN THIS PLATFORM CAN EASILY SAVE DRUG DEVELOPMENT COST THROUGH FIVE YEARS IN DRUG DEVELOPMENT. LASTLY, THIS IS IS WELL SUPPORTED AT NCATS WITH EFFICIENT PROGRAM MANAGERS AND WE HAVE A NUMBER OF SUPPORT FROM A NUMBER OF NIH INSTITUTES AND CENTERS AS WELL AS COLLABORATIONS WITH NASA AND DARPA AND AS WELL AS THE I.Q. CONSORTIUM AND WITH THAT I'LL END AND HAPPY 10th BIRTHDAY NCATS. >> THANK YOU SO MUCH FOR YOUR TALK. I WANT TO THANK ALL OF OUR SPEAKERS. WE HAD INCREDIBLE CONVERSATION. I KNOW WE'RE ALL PROUD OF THE WORK HAPPENING AT NCATS. I KNOW WE'RE RUNNING A LITTLE BIT OVER TIME. A COUPLE QUICK POINTS IN TERM OF WHAT WE HEARD DURING THIS SESSION IMPORTANTLY THE WORK HAPPENING TO MAKE SURE WE OVERCOME THE VALLEY OF DEATH AND MAKE SURE WE HAVE EFFECTIVE PROJECT TEAMS AND FINDING BETTER WAYS TO DO OPEN SCIENCE AND THE VARIOUS PLATFORM TECHNOLOGIES TO TEST NEW COMPOUNDS AND THE FUTURE OF TISSUE CHIPS AND EFFICACY AND COVID-19 CREATED A CATALYTIC MOMENT TO ADDRESS THE ISSUES IN DRUG DEVELOPMENT AND ACCELERATE US TO THE FUTURE PARTICULARLY AS WE LOOK DEEPLY INTO THE RARE DISEASE CONDITIONS. I'M INCREDIBLY THRILLED TO SEE THE WORK THAT'S HAPPENED OVER THE 10 YEARS AND LOOK FORWARD TO THE WORK AHEAD. THANK YOU ALL SO MUCH. >> MY NAME IS CHRISTINA HARTMAN AND I ALSO SERVE AS A MEMBER OF THE NCATS ADVISORY COUNCIL. I'M ON THE BOARD OF GLOBAL GENE A NON-PROFIT DEDICATED TO ELIMINATING THE BURDENS AND CHALLENGES OF RARE DISEASES FOR PATIENTS AND THEIR FAMILIES. I'M ALSO ON THE BOARD OF THE FOUNDATION FOR THE FORMER ARMED FOUNDATION A NON-PROFIT DEDICATED TO PROVIDING DEDICATION, INFORMATION AND RESEARCH TO ACCELERATE PATIENT AWARENESS OF AND ACCESS TO TRANSFORMATIVE THERAPIES. MOST IMPORTANT I'M A RARE MOM. MY YOUNGEST DAUGHTER HAS AN YOU WILL ULTRA RARE CONDITIONS DISORDER. THE GENE ENCODES FOR AN ESTIMATED 40% TO 50% OF PROTEINS AND THEIR BRAINS ARE NOT PROCESSING APPROPRIATELY AND MAKING IT DIFFICULT FOR THEM TO FUNCTION INDEPENDENTLY. WE NEED ACCESSIBLE TREATMENT. AS DR. COLLINS STATED WE NEED TO FIGURE OUT HOW TO TAKE THE SCIENCE AND POUR IT INTO ACTION IN THE CLINIC WOULD CROSSING THE DIFFICULT TERRITORY OFTEN CALLED THE VALLEY OF DEATH AND BASIC SCIENCE IS OFTEN TOO RISKY FOR COMMERCIAL DEVELOPMENT. WITH 10,000 RARE DISEASES 95% OF WHICH DID NOT HAVE AN FDA APPROVED TREATMENT WE MUST INVEST IN NCATS AND TRANSLATIONAL MEDICINE. WE DON'T HAVE TIME FOR THE USUAL ONE-OFF APPROACH OF PURSUING A TREME TREATMENT FOR A DISEASE. IT WILL NOT WORK AND IN RECENT YEARS WE'VE HAD MAJOR WINS THE COVID-19 VACCINE WAS CREATED AND THE mRNA VACCINES ARE PLUG AND PLAY AND MAKE A PROTEIN TO TRIGGER AN IMMUNE RESPONSE IN SIDE OUR BODY. GENE THERAPIES APPROVED FOR MUSCULAR ATROPHY USE AN ADENO ASSOCIATED VIRUS VECTOR TO DELIVER THE GENE THERAPY. AAV MANUFACTURING CAPACITY REMAINS A CHALLENGE PARTICULARLY FOR SMALL SCALE LOTS AND RARE DISEASE CLINICAL TRIALS AND MANY ARE WAITING FOR THIS PLATFORM TO BE USED IN THEIR DISEASES AND THE FIRST PERSON IN THE WORLD RECEIVED THE DRUG FOR JUST ONE PERSON AND THE WORLD LOST HER BUT THE RESEARCHER AND HER MOM REMAIN ON AN A MISSION FOR THE IDEA TO AN INJECTION IN LESS THAN A YEAR TO AN ENTIRELY NEW TREATMENT PATH OF PERSONALIZED MEDICINE ACCESS ABLE ACROSS ALL RARE DISEASES. I'D LIKE TO INTRODUCE EXPERTS THE RARE DISEASE COMMUNITY IS GRATEFUL TO HAVE IN OUR CORNER AND PAVED THE WAY FOR DEVELOPMENT OF NEEDED TREATMENTS AND CURES. THE FIRST SPEAKER DR. ED NEILAN IS AT THE NATIONAL ORGANIZATION FOR RARE DISORDERS. THROUGHOUT HIS CAREER HE HAS CARED FOR MANY RARE DISEASE PATIENTS AND CONTRIBUTED TO THE REGULATORY APPROVAL OF FIVE NEW RARE DISEASE THERAPIES. OUR SECOND SPEAKER MATTHEW HALL HAS RESEARCH AND DEVELOPMENT WORK INCLUDING IDENTIFYING THERAPEUTIC CANDIDATES TO REPURPOSE FOR RARE DISEASES AND WORKS ON THE NIH COVID RESPONSE EFFORT. OUR THIRD SPEAKER DR. PJ BROOKS IS THE DEPUTY DIRECTOR OF THE NCATS OFFICE OF RARE DISEASE AND APART OF PILOT PROJECTS. OUR FOURTH SPEAKER, DR. PETER MARKS IS FROM THE BIOLOGIC EVALUATION AND RESEARCH AND TAUGHT AND CARED FOR PATIENTS AND WORKED WITH INDUSTRY AND DRUG DEVELOPMENT AND IS AN ARCHITECT. I'LL NOW TURN THINGS OVER TO ED. >> YEARS BEFORE BEFORE I COMMITTED MYSELF TO A CAREER COMBINING MEDICINE AND SCIENCE I READ THE BOOK THE YOUNGEST SCIENCE NOTES OF A WATCHER BY A POP YOU -- POPULARIZER OF SCIENCE AND NOTED THE PHARMACO AT THAT TIME INCLUDED ONLY A HANDFUL OF TRULY EFFECTIVE MEDICATIONS. AND THAT AMONGST THE MANY ELIXIRS THEN PRESCRIBED FOR VARIOUS AILMENTS OFTEN WITHOUT SCIENTIFIC EVIDENCE THE MOST POPULAR TEND TO BE IN HIS ESTIMATION THOSE THAT CONTAIN THE MOST ALCOHOL. AND IN THE CONTEXT OF THIS SESSION WE SHOULD NOTE PENICILLIN IS ONE DRUG USEFUL FOR MULTIPLE DISEASES HAVE BEEN IN COMMON IN INFECTION BY BACTERIA THAT HAVE A CERTAIN KIND OF CELL WALL STRUCTURE. AND UP TO THAT TIME DRUGS INCLUDING PENICILLIN WERE DISCOVERED THROUGH A HAPPY ACCIDENT AND THEN A MULTITUDE OF DRUGS WERE DISCOVERED THROUGH PURPOSEFUL AND RANDOM SCREENING OF DOZENS TO THOUSANDS OR MILLIONS OF CHEMICAL COMPOUNDS AND TO THIS DAY SCREENING AND DRUGS TO FIND A COMPOUND WITH DESIRED ACTIVITY REMAINS IMPORTANT TO DRUG DISCOVERY AS YOU'LL HEAR LATER IN MATTHEW HALL'S TALK. WE ENTERED INTO A NEW ERA NOW WITH THE VAST EXPLOSION OF KNOWLEDGE OF GENETICS AND DEVELOPMENT OF MULTIPLE CLASSES OF GENE-TARGETED THERAPIES AND THE ORIGINAL CONCEPT OF GENE THERAPY BY THERAPEUTIC GENE REPLACEMENT AND RNAI AND VARIOUS GENE EDITING APPROACHES MAKING IT POSSIBLE FOR MANY DISEASES TO DESIGN A THERAPY ON PAPER OR AT LEAST ON A COMPUTER FROM A GENE SEQUENCE AND HAVE A GOOD CHANCE OF THE DESIGN THERAPY OR A SLIGHT VARIATION OR A RECEPTIVE GENETIC DISEASE YOU CAN DESIGN A GOOD GENE REPLACEMENT THERAPY WITHOUT KNOWING WHAT IT DOES OR WHAT THE DOWNSTREAM STEPS ARE IN THE PATHOGENESIS OF THAT DISEASE. AND THE LIMITING FACTOR IS NO LONGER DEVELOP IING DISCOVERING A ONE IN A MILLION DRUG BUT RATHER MORE OPERATIONAL HURDLES. COME COULD BE ADDRESSED THROUGH PLATFORM APPROACHES AS YOU'LL HEAR MORE ABOUT IN P.J. BROOKS' UPCOMING TALK. AND FOR RARE DISEASES TO THE NATURAL HISTORY DATA IS MISSING. AND THE EVOLUTION IN DRUG DEVELOPMENT COINCIDES WITH THE SHIFT IN THE ROLE OF PATIENT ORGANIZATIONS WHICH ARE NOW IN A BETTER POSITION TO PUSH FORWARD THERAPY DEVELOPMENT EFFORTS. FIRST, PFSHT GROUPS WORKING WITH OR ASSISTED BY NCATS THE GREAT EXAMPLE OF ENABLING PATIENT GROUPS AND DRIVING FORWARD THERAPEUTIC DEVELOPMENT IS THE NCATS TOOL KIT FOR PATIENT FOCUSSED DEVELOPMENT. IT'S ONLINE INFORMATION INCLUDING LINK TO OUTSIDE RESOURCES THAT PROVIDE GUIDELINES TO UNDERSTAND THE DRUG DEVELOPMENT PROCESS AND WORK WITH STAKEHOLDERS TO VANCE DRUG DEVELOPMENT THROUGH THE PROCESS. AND SOME OF THE NCATS TOOL KIT LINKS POINT TO RESOURCES. ANOTHER GREAT NCATS RESOURCE FOR PATIENT GROUPS IS RADAR AND WEBSITE THAT PROVIDES PATIENT GROUPS WITH GUIDANCE HOW TO DEVELOP REGISTRIES FOR RARE DISEASES. GOOD QUALITY REGISTRIES HELP SUPPORT RESEARCH PROGRAMS AND STIMULATE TREATMENT DEVELOPMENT. AND THERE'S PROGRAMS TO SUPPORT PATIENT OWNED REGISTRIES IN NATURAL HISTORY STUDIES AND DEVELOPING AND HOSTING ON THE SOFTWARE PLATFORM A GROWING NUMBER OF RARE DISEASE REGISTRIES AND NATURAL HISTORY STUDIES EACH OF WHICH IS TYPICALLY OWED BY PATIENT ORGANIZATION AND CO-DESIGNED WITH THEM AND PATIENT GROUPS WORKING WITH RESEARCHERS. THERE'S A GROWING NUMBER OF SUCCESSFUL EXAMPLES OF PATIENTS WORKING WITH PATIENTS TO DRIVE THE DEVELOPMENT OF THERAPEUTICS. A GREAT ONE IS THE PROGERIA FOUNDATION WHICH FIRST RECRUITED FRANCIS COLLINS TO HELP IDENTIFY THE GENE AND RECRUITED ACADEMICS AT BOSTON CHILDREN'S HOSPITAL TO HELP CONDUCT CLINICAL TRIALS AND THE COLLABORATIONS LED TO THE 2020 FDA APPROVAL OF THE TRANSFERASE INHIBITOR FOR THE TREATMENT OF THE PROGERIA AND OTHER LAMINOPATHIES AND THERE'S THE NCATS RUN RDRCN OR RARE DISEASE CLINIC RESEARCH NETWORK WHICH FUNDS AND ENABLES RESEARCH CONSORTIA EACH OF WHICH MUST HAVE AT LEAST ONE PATIENT ADVOCACY ORGANIZATION INVOLVED AND EACH OF WHICH TAKES MANY DISEASES AT A TIME APPROACH FOSTERING RESEARCH IN A PARTICULAR DISEASE AREA. THIS LED TO MANY VANCE -- ADVANCES AND THEY DEVELOPED END POINTS BY THE LYSOSOMAL DISEASE NETWORK. THIRD, PATIENTS WORKING DIRECTLY WITH DRUG COMPANIES. PATIENT GROUPS ARE INCREASINGLY WORKING WITH PHARMACEUTICAL COMPANIES. FOR EXAMPLE DURING MY TIME WOMAN A COMPANY WE WORKED AND A PROJECT ASSISTED BY ACADEMIC NEUROLOGISTS COLLECTED DATA INCLUDING TESTING POSSIBLE CLINICAL TRIAL END POINTS WITH LATE ONSET 2 BY ATTENDING SUPPORT GROUP MEETINGS OF THE ALLIED DISORDERS ASSOCIATION AND PERFORMING THE CLINICAL ASSESSMENTS IN SATELLITE ROOMS DURING THE MEETINGS AND THE DATA COLLECTED OVER FIVE ANNUAL MEETINGS PROVIDED ENOUGH INFORMATION TO HELP DEAN A PHASE 3 TRIAL IN LATE ONSET GM2. AND FOLLOWING A MANY DISEASE IN ONE APPROACH, THAT TRIAL ALSO INCLUDES OTHER GLYCOLIPIDS. FOURTH, AS AN EXAMPLE A VERY PROMISING PUBLIC PRIVATE PARTNERSHIPS AND THE CONSORTIUM WAS MENTIONED EARLIER BY DR. COLLINS AND WILL BE COVERED IN MORE DETAILS DURING THE TALK LATER IN THE SESSION. FINALLY, AS MANY MAY BE AWARE, LAST YEAR THERE WAS AN ANNOUNCEMENT OF THE RARE DISEASE CENTERS OF EXCELLENCE IN LEADING MEDICAL INSTITUTIONS ACROSS THE U.S. BASICALLY THE CHALLENGE OF TRYING TO IMPROVE CARE IN THOUSANDS OF RARE DISEASES MOST OF WHICH HAVE NO SPECIFIC FDA TREATMENT WILL BE WORKING WITH OUR PARTNERS ON COLLABORATIVE PLATFORM APPROACHES AIMED TO BENEFIT BENEFIT RARE DISEASE COMMUNITIES SIMULTANEOUSLY. WITH THAT I'LL PASS IT TO DR. MATTHEW HALL OUR NEXT SPEAKER. THANK YOU FOR YOUR ATTENTION. >> AND I'M PASSIONATE ABOUT EVERYTHING WE'RE HEARING ABOUT TODAY AND IN THE APPROACH OF TRYING TO TREAT MANY DISEASES AT A TIME. AS ED TOUCHED ON WHEN YOU THINK OF NCATS PEOPLE THINK ABOUT RESEARCH LABS AND BIG YELLOW ROBOTS IN THE HIGH THROUGHPUT SCREENING AND WE OVER THE YEARS TESTED OR CONDUCTED OVER 600 MILLION DIFFERENT EXPERIMENTS WITH DRUGS AND DISEASES HERE AT NCATS. IT'S BEEN A REAL ENGINE ROOM AND POWERHOUSE IN TESTING AS MANY COMPOUNDS AGAINST AS MANY DISEASES AS POSSIBLE IN TRYING TO WORK WITH OTHERS IN THE RESEARCH COMMUNITY TO FIND CURES FOR DISEASES AND A BIG PART OF THAT FOR US AND CHRISTINE TOUCHED ON IS THE POWER OF DRUG REPURPOSING AND THE OPPORTUNITY IT PRESENTS. IF START FROM SCRATCH FROM THE DRUG DEVELOPMENT PROGRAM YOU CAN TAKE HUNDREDS OF THOUSANDS OF COMPOUNDS AND TAKE UP TO 15 YEARS TO DEVELOP THE THERAPEUTIC AND GET IT IN THE CLINIC AND COSTS AN INCREDIBLE AMOUNT OF MONEY AND IT'S NOT IDEAL FOR THE AMOUNT OF DISEASES AND REPURPOSING OFFERS A PATHWAY SO AT NCATS WE CREATED A LIBRARY OF NEARLY 3,000 DRUGS APPROVED IN THE UNITED STATES IN EUROPE AND JAPAN AND OTHER PLACES AND THE COMPOUNDS ARE IN THE NCATS PHARMACEUTICAL COLLECTION DEVELOPED AS NCATS WAS BORN OV10 YEARS AGO. WE KNOW ABOUT THE COMPOUNDS AND DRUGS AND HOW THEY WORK AND HOW TO ADMINISTER THEM AND THEY'RE SAFE AND PROVIDE A SPEEDY PATHWAY AND THE PATHWAY TO THE CLINIC GOES FROM 15 YEARS TO LESS THAN A YEAR. ON TOP OF THAT WE SAW LAST YEAR AND WITH COVID THE DRUG REPURPOSING TRIAL COULD BE INITIATED AND EXECUTED IN A MATTER OF WEEKS. THE DRUG REPURPOSING LIBRARY WE SCREENED OVER 800 TIMES AND THE FIRST THING WE DO IN DEVELOPING A NEW DISEASE IS UPDATE THE LIBRARY EVERY YEAR AND WE KNOW FOR EVERY DRUG APPROVED MANY DRUGS HAVE BEEN IN CLINICAL TRIALS AND FAILED AND SOMETHING WE TRY TO IMPROVE OR SOLVE THE DRUG FAILURE AND WE'RE WORKING TO CREATE A LARGER LIBRARY BEYOND JUST DRUGS APPROVED BUT EVER BEEN IN HUMANS TO EXPAND THE NUMBER OF POSSIBILITIES FOR TRYING TO IDENTIFY DRUG REPURPOSING OPPORTUNITIES FOR PATIENTS WITH RARE DISEASE. WE HAVE ALL THIS KNOW HOW AND EXPERIENCE WITH DRUG REPURPOSING AND BRINGING TOGETHER THE RIGHT BIOLOGICAL EXPERIMENT TO TEST THE APPROVED DRUGS AGAIN AND SARS COV2 CAME LONG AND -- ALONG AND IT WAS A BRAND NEW DISEASE AND THERE WERE NO THERMOMET THERAPEUTICS AND WE NEEDED TO UNDERSTAND THE VIRUS AND WORK OUT THE ASSAYS AND THE EXPERIMENTAL APPROACHES AND NEEDED TO UNDERSTAND WHAT COMPOUNDS MAY WORK AND ALL INSTANTLY AND IN PARALLEL. WHAT WE DECIDED TO DO AT NCATS WAS BROUGHT TOGETHER THE VISION WE SHOULD TRY AND TEST AS MANY DRUGS AND MOLECULES AS WE POSSIBLY CAN AGAINST AS MANY COVID ASSAYS AND MAKE THAT AVAILABLE TO THE WORLD IMMEDIATELY NOT THINKING ABOUT THINGS SCIENTISTS THINK ABOUT LIKE PUBLICATION TIME LINES AND GET TE DATA OUT. WE DROPPED EVERYTHING AND DEVELOPED ULTIMATELY OVER 30 DIFFERENT BIOLOGICAL ASSAYS TO SCREEN DRUG COLLECTIONS AGAIN EVERYTHING FROM EXPERIMENTS WITH PROTEINS TO WORKING WITH LIVE VIRUS AND A LOT OF THINGS IN BETWEEN. AS WE DEVELOPED THE OPEN DATA PORTAL WE PUT ACTIVITY DATA AS WE GENERATE IT ALMOST IMMEDIATELY AND AVAILABLE FOR EVERYONE TO LOOK AT AND DOWNLOAD AS WELL. AS TIME WENT ON WE PUT ON EXPERIMENTAL PROTOCOLS SO ANYONE COULD REPLICATE THOSE IN THE WORLD AND INFORMATION ABOUT THE BEST ANIMAL MODELS TO USE AND THE PLACE WHERE WE TRACK ALL VARIANTS AS PART OF OUR WORK WITH THE NIH ACTIVE PROGRAM AND THAT IS A DAILY UPDATE EVEN THIS MORNING WE LOOKED AT THE FIRST OMNICRON DATA AND LOOKING TO GET IT IN THE PORTAL AS QUICKLY AS POSSIBLE. LATER ON IN 2020 WE ORGANIZED A SUMMIT AND WHERE WE STAND AND WHAT WE HAVE AND WHAT'S IN THE DEVELOPMENT PATHWAY AND PUT TOGETHER PHARMA PARTNERS FROM ACROSS THE GLOBE, ACADEMICS, GOVERNMENT SCIENTISTS AND OTHERS TO EVALUATE WHAT WE HAVE AND WHAT WE NEED. A LESSON THAT CAME WAS THINGS WERE UNDERWAY IN TERMS OF DRUG V AND WE RECOGNIZED THERE WERE NO CORONAVIRUS DRUGS BEFORE SARS COV2 AND MAYBE WE SHOULD THINK ABOUT HOW TO PREPARE FOR THE FUTURE THE NEXT TIME IT'S HAPPENED. THERE'S GOOD REASON FOR THAT. WE'VE HAD IN OUTBREAKS OVER THE PAST TWO DECADE. NONE OF THEM BECAME PANDEMICS BUT SEVERAL HAVE THREATENED. WE KNOW ABOUT SARS AND MERS AND VIRUSES LIKE CHI GEN -- CHIKUNGUNYA AND WE HAVE VIRUS THREATEN TO JUMP FROM ANIMAL TO HUMAN OR BREAK OUT AS A PANDEMIC. FIRST THE DISEASES DON'T EXIST YET BUT WE KNOW THE THREAT AND WE CAN BE PREPARED FOR FUTURE PANDEMICS BUT NCATS COMES IN AND THE NIH ENVIRONMENT COMES IN WE CAN CATALYZE THE DEVELOPMENT FOR PATIENTS WITH RARE DISEASES AND MAYBE FUTURE DISEASES AS WELL. FOR ANTIVIRALS WE DECIDED TO TRY TO BUILD A PROGRAM AND DR. COLLINS THANKFULLY SPARK TE IDEA OF PUTTING TOGETHER A LARGE PROGRAM TO CREATE THE ANTIVIRALS ACTIVE AGAINST MANY DIFFERENT VIRUSES IN PILL FORM AND AREN'T REALLY AVAILABLE AND COULD BE ADMINISTERED ANYWHERE IN THE WORLD AND IF YOU HAVE THE TOOLS AVAILABLE YOU SHOULD BE ABLE TO SAVE LIVES, REDUCE SERIOUS ILLNESS AND PREVENT SURGES IN HOSPITALIZATION AND BETWEEN CHRISTMAS AND NEW YEAR WE WORKED WITH DR. COLLINS OFFICE AND WROTE A PROPOSAL TO CREATE THERAPEUTICS AGAINST ALL FUTURE THREAT VIRUS TO PREVENT AND BE READY FOR THE FUTURE PANDEMIC WHEN IT CURBS. -- WHEN IT OCCURS. WE WERE LUCKY ENOUGH TO SEE THE PROGRAM FUNDED THROUGH THE AMERICAN RESCUE PLAN ACT AND AT THE MOMENT IT'S OVER $3 BILLION OF INVESTMENT OVER FIVE YEARS WITH THE GOAL OF THE ANTIVIRALS FROM EARLY DISCOVERY THROUGH LATE STAGE DEVELOPMENT AND HAVE SAFE FORMULATED MEDICINES WITH NO ACTIVITY AVAILABLE SO WHEN THE TIME COMES AND WE NEED TO IDENTIFY TREATMENT OPPORTUNITIES FOR PEOPLE ANYWHERE IN THE WORLD WE'LL HAVE THOSE AT OUR PROPOSAL. YOU MAY NOT KNOW THE KIND OF VIRAL FAMILIES OR HEARD OF THE ONES ENCOMPASSED WITHIN THE PROGRAM BUT WHEN I MENTIONED VIRUSES LIKE ZIKA OR EBOLA IT GIVES YOU THE IDEA OF THE THREAT VIRUS EXIST. NCATS WITH ALL OF OUR EXPERIENCE AND WE'VE BOTH DONE MANY DIFFERENT VIRUSES OVER THE YEARS AND RESPONDED TO EMERGENCIES LIKE EBOLA AND ZIKA AND MERS WHEN THEY OCCURRED AND PUT TOGETHER A PROGRAM WHERE WE'LL BE PARTNERING WITH PEOPLE IN BIO TECH AND PHARMA AND END ACADEM -- ACADEMIA AND TAKE DRUGS TO CLINICAL TRIALS AND SHOW THEY'RE SAFE SO WE'LL BE READY FOR THE NEXT PANDEMIC WHEN IT COMES ALONG. THE KEY THING WITH TO THE ANTIVIRALS IS WE CREATE THEM ONCE AND WE CAN TREAT MANY INFECTIOUS DISEASES IN THE WORLD WHETHER THEY EXIST OR NOT. THAT PREPARATION IS CRITICAL. MY HOPE IS WHEN WE GET TO NCATS 20th ANNIVERSARY WE CAN SAY WE WERE SUCCESSFUL AT THIS AND TAKE THE LESSONS OF HOW WE RESPONDED TO COVID-19 AND HOW WE WERE ABLE TO PUT TOGETHER THE PLATFORMS AND APPLY THEM TO CHRONIC AND RARE DISEASE AND TAKE THE LESSONS AND APPLY THE LESSONS MANY TIMES AS WELL. AGAIN, IT'S A REAL HONOR TO BE HERE AND I'LL PASS IT TO MY COLLEAGUE D.J. BROOKS AT NCATS. >> THANK YOU. TO ME WHEN I THINK OF MANY DISEASE AT A TIME WHAT IT MEANS SO ME IS THOUGH THERE'S THOUSANDS OF DISEASES THERE'S A RELATIVELY SMALL NUMBER OF CAUSE OR IDEOLOGY AND THEY CAN BE TARGETED BY DRUGS OR BUY LIOLOGICS AND WE CAN GET MORE TREATMENT TO MORE PATIENTS AS SOMEONE WHO WORKS FOR THE OFFICE OF RARE DISEASE RESEARCH BUT THE IDEA IS NOT REALLY LIMITED AND THERE'S BACTERIAL INFECTIOUS DISEASES AND THE UNDERLYING IDEOLOGY IS A BACTERIAL INFECTION CAN BE TARGET ANTIBIOTICS. SAME FOR VIRUSES. A PARTICULAR RELEVANCE IN THE ONCOLOGY WE HAVE AGNOSTIC BASKET TRIALS THAT TARGET MOLECULAR PATHWAYS THAT HAVE LED TO DRUG APPROVAL IN THE FDA. WITH REGARD TO RARE DISEASES THERE'S THOUSANDS OF THEM AND THERE'S A LIMITED UNDER OF ETIOLOGY SOME OF WHICH ARE DRUGGABLE. WE OUGHT TO BE ABLE TO TAKE THIS APPROACH AND APPLY IT TO RARE DISEASES AND IN THAT REGARD WE PUT OUT A FUNDING OPPORTUNITY TO SUPPORT CLINICAL TRIALS IN RARE DISEASES WITH THE GOAL OF ADAPTING THE BASKET TRIAL APPROACH INTO THE RARE DISEASE SPACE AND WE FUNDED TWO OF THESE PROJECTS AND STILL HAVE AN OPEN FO A FOR SMALL BUSINESSES AND I THINK THIS IS AN AREA WE WANT TO CONTINUE FOCUSSING ON. THERE'S SOME DISEASES SO RARE NO ONE WOULD EVER OPEN UP A CLINICAL TRIAL FOR THEM DESPITE THE PATIENTS WITH THE DISEASES WOULD RESPOND TO DRUGS AVAILABLE AND NEED TO GET THEM INTO CLINICAL TRIALS. AND RARE MONOGENIC DISEASES PRESENT A CLEARER OPPORTUNITY FOR MANY DISEASE AT A TIME APPROACH BECAUSE BY DEFINITION THESE ARE CAUSED BY VARIANTS THAT INACTIVATE A SINGLE GENE. IF YOU CAN PUT A WORKING COPY OF THAT GENE TOO THE TARGET CELLS FOR THE DISEASE AT THE APPROPRIATE TIME OF DEVELOPMENT AND DISEASE PROGRESSION YOU COULD WE WILL HAVE A BENEFICIAL CLINICAL EFFECT AND IT'S POSSIBLE TO DO THIS FOR CELLS AND TISSUES USING GENE THERAPY WITH ADENO ASSOCIATED VIRUSES OR AEV. IT'S REALLY NOT A DISEASE SPECIFIC TREATMENT IT'S A THERAPEUTIC PLATFORM AND DEVELOP THEM TO CELLS AND TISSUES. WE'VE NOW SEEN SOME SOME PROOF OF CONCEPT STUDIES IN ANIMAL MODELS. THE REAL PROBLEM IS GETTING SOME INTO CLINICAL TRIALS. AND UNFORTUNATELY THE CURRENT APPROACH TO AAV GENE THERAPY IS TO FOCUS ON ONE DISEASE AT A TIME. AND THAT APPROACH DOESN'T TAKE COMPANIES WILL TEND TO FOCUS ON THE MOST COMMON RARE DISEASES AND SOME COULD BE LEFT AND OFTEN FAMILIES WILL HAVE TO SUPPORT THESE TRIALS. WE STARTED IT IN AN EFFORT FOR THE START UP. THE IDEA IS WE USE THE SAME VECTOR FOR FOUR DIFFERENT DISEASES, USE THE SAME MANUFACTURING MATERIAL AND CHANGE THE THERAPY TO GENE ITTED SHOULD BE POSSIBLE TO REMOVE THE STEPS NECESSARY TO GET IT STARTED LIKE THE BIO DESCRIBE USING STUDIES OR OTHER THINGS FOR THAT MATTER AND WE WERE PROPOSING REFINEMENTS TO THE FDA ON OUR BASED ON THE PLATFORM APPROACH AND IMPORTANTLY WE PLAN TO ASK THESE QUESTIONS OF THE FDA AND TAKE RESPONSES BACK AND MAKE IT PUBLICLY AVAILABLE WITH DOCUMENTS AND IMPROVED INDs ULTIMATELY WITH THE IDEA OF BENEFITING FUTURE GENE THERAPY CLINICAL TRIALS PARTICULARLY OF DISEASES OF NO COMMERCIAL INTEREST AND THIS IS BEING DONE WITH THE NHGI AND OTHERS. IN A SIMILAR VEIN I'VE REPRESENTED NCATS AND THE NIH IN THE GENE THERAPY CONSORTIUM OR BGTC REFERRED TO EARLIER. IT'S ALSO FOCUSSED ON INCREASING THE EFFICIENCY OF AAV GENE CLINICAL TRIALS AND STREAMLINE THE REGULATORY PATHWAY. THEY ALSO EFFORT TO IMPROVE MANUFACTURING EFFICIENCY AND CLINICAL BENEFIT. HOWEVER, IN CONTRAST THE BGT IS A PUBLIC PRIVATE PARTNERSHIP INVOLVING COMPANIES AND INSTITUTES AND CENTERS AND NON-PROFIT ENTITIES AND THE FDA CENTER FOR BIOLOGICS. IT'S AN ACCELERATED MEDICINE PROGRAM ORGANIZATIED BY THE FNIH AND SUPPORTING THE PUBLIC PRIVATE PARTNERSHIPS WE COULDN'T DO AS NIH EMPLOYEES AND PROVIDE AS A VENUE FOR COLLEAGUES AND THE SCREENING GROUP TO BE INVOLVED TO WORK WITH INDUSTRY SPECIFICALLY IN THIS PRE-COMPETITIVE SPACE AND THERE'S REAL OPPORTUNITY TO MAKE PROGRESS. I WANT TO THANK AND ACKNOWLEDGE THE EFFORTS OF DR. PETER MARKS FOR HIS EFFORT ESPECIALLY AT A DIFFICULT TIME IN THE CONTEXT OF THE COVID PANDEMIC AND WE RECENTLY RELEASED FUNDING OPPORTUNITIES AS WELL AS A CALL FOR DISEASE FOM -- NOMINATIONS. AND FINALLY THE BROADEST TREATMENT FOR TREATING MONOGENETIC DISEASE AND GENOME EDITING EM EM PLIFIDE BY CRISPR CAS 9 AND ARE YOU DELIVER THE ENZYME INTO CELLS WHICH CAN DIRECTLY CORRECT DISEASE CAUSING MUTATIONS AND THEY'RE INVOLVED IN COORDINATION OF THE COMMON FUND PROGRAM AND SOMATIC CELL GENE EDITING TOOLS AND BETTER ET FORS AND DELIVERY SYSTEMS AND ANIMAL MODELS FOR CELLS AND TISSUES. IT'S WORTH NOTING SOME OF THESE DELIVERY VEHICLES USE NANO PARTICLES THE SAME DELIVERY SYSTEM AS COVID-19 AND IS THE RARE BENEFIT OF THIS OTHERWISE TERRIBLE PANDEMIC. WE RECENTLY OBTAINED CONCEPT CLEARANCE TO MOVE TO THE NEXT PHASE FOR CLINICAL IMPLEMENTATION INCLUDING CLINICAL GENOME EDITING TRIALS REQUIRED TO STUDY MORE THAN ONE DISEASE AT A TIME TO EMBRACE THE PLATFORM EDITING OF GENOME EDITING AND THE PLATFORMS ARE TO ENABLE TO TREAT MANY DISEASES RARE AND COMMON. AND SO WITH THAT I'M GOING TO STOP AND TURN IT OVER TO PETER MARKS. >> THANK YOU VERY MUCH, P.J. HE COVERED A LOT OF WHAT IS GREAT TO LET ME GET SET AND TALK ABOUT WHAT I WOULD CONSIDER MANY DISEASES AT ONCE. AND FIRST OF ALL, THE WORK AT NCATS WAS A NATURAL PARTNERSHIP TO TRY TO MOVE FORWARD TO HELP ADVANCE GENE THERAPIES FOR RARE DISORDER. I'M VERY GRATEFUL TO P.J. AND HIS COLLEAGUE FOR WORKING TOGETHER TO HELP PUT SOMETHING TOGETHER WITH THE CONSORTIUM WHICH HAS BEEN LAUNCHED AS A PROJECT AND TO HELP US MOVE FORWARD AND ADDRESS A PROBLEM THAT IS REALLY THERE THAT IS ENIGMATIC. WE HAVE GENE THERAPIES WHERE WE'VE SEEN REMARKABLE TRANSFORMATIONS OF DISEASE ENTITIES LIKE SMA TYPE 1 AND YET WE HAVE MANY MANY OTHERS THAT ARE JUST SIMPLY NOT BEING ADDRESSED BECAUSE THERE'S JUST NOT COMMERCIAL VIABILITY AS ALLUDED TO. THIS CONCEPT OF BEING ABLE TO RE-USE AND LEVERAGE OUR KNOWLEDGE SO THAT EACH TIME WE'RE NOT STARTING FROM SCRATCH WITHGENE -- WITH GENE THERAPIES HAS BEEN IMPORTANT TO EXPLORE. THAT'S ONE OF THE PURPOSES OF THE THERAPY CONSORTIUM. HOW DO YOU LEVERAGE AS MUCH AS YOU CAN OUT OF ADENO ASSOCIATED VIRUS AND IN TERMS OF THE BIOLOGY OF ADENO ASSOCIATED VIRUS AND HOW DO YOU LEVERAGE AS MUCH AS YOU CAN OUT OF THE MECHANICS OF ADENO ASSOCIATED VIRUS? THAT IS, YOU'VE DECIDED YOU HAVE A VECTOR YOU'RE NOW GOING TO USE SPECIALLY REPEATEDLY TO SHUTTLE IN DIFFERENT INSERTS TO DIFFERENT RARE DISEASES. THERE'S DIFFERENT TYPES OF AAVs. THAT'S OKAY. FOR ANY GIVEN AAV TYPE THE IDEA IS IF WE CAN START TO THINK ABOUT HOW TO REUSE THE VECTORS AND TREAT THEM ALMOST A LITTLE BIT LIKE DEVICES RATHER THAN THE AS WE TYPICALLY THINK OF GENE THERAPY VECTORS IF WE THINK OF THE VECTOR THAT'S HOLDING THE INSERT THAT WAY, WE POTENTIALLY COULD START TO LEVERAGE THINGS MORE. OBVIOUSLY, THERE'S A LOT OF CAVEATS ON THAT BECAUSE THE VECTOR IS NOT A DEVICE IT'S A BIOLOGIC STILL BUT THE CONCEPT IS NOT REINVENTING THE WHEEL WHEN WE ENGINEER FOR A NEW RARE DISEASE. THE OTHER PIECE THAT'S IMPORTANT IN THE BESPOKE GENE THERAPY CONSORTIUM IS THE CONCEPT THAT ULTIMATELY IF WE DO THINGS RIGHT WE'LL UNDERSTAND HOW TO NOT ONLY LEVERAGE THE VECTORS BUT ULTIMATELY HAVE A COOKBOOK A MANUAL FOR HOW TO DO THIS SO THAT EVERY TIME AN INVESTIGATOR IN AN ACADEMIC OR COMPANY HAS A PLAY BOOK TO FOLLOW TO GO DOWN A ROAD TRAVEL PREVIOUSLY. THAT'S LIKELY TO GET US MOST QUICKLY TOWARDS INNOVATION RATHER THAN REINVENTING THE BASICS EVERY TIME. IN A WAY IT'S BASICALLY ALLOWING ONE TO NOT HAVE TO FIGURE OUT HOW DO I GROW OUT MY CELLS AND GET MY VECTOR PURIFIED EACH TIME AND USING DIFFERENT PROTOCOLS THAT HOPEFULLY COULD BE THOSE DIFFERENCES COULD BE DEALT WITH BY HAVING SUCH A COOKBOOK OR GUIDE MANUAL. ULTIMATELY THE COMBINATION OF THINGS IS PUT TOGETHER AND THE HOPE IS THAT WE WORKING TOGETHER WITH NCATS UNDER THE OFFICES OF THE FOUNDATION OF NIH ALONG WITH COMPANIES AND NON-PROFIT AGENCIES AND ADVOCACY GROUPS CAN PUT TOGETHER A MODEL SO IF YOU HAVE A MODEL WHEREBY YOU CAN APPROPRIATELY LEVERAGE THESE GENE THERAPY VECTORS AND APPROPRIATELY LEVERAGE PREVIOUS KNOWLEDGE HOPEFULLY THE COST OF MAKING A GENE THERAPY FOR INDIVIDUALS WILL COME DOWN SIGNIFICANTLY SO THIS BECOMES A COMMERCIALLY VIABLE OPPORTUNITY AND THAT COMMERCIAL VIABILITY IS CRITICAL IF WE'RE GOING TO START TO SEE HUNDREDS OF RARE DISEASES EQUITABLY ADDRESSED. THE GOAL IS TO HAVE IT BE SUCH THAT WE DON'T HAVE TO WORRY THERE'S DISEASES THAT ARE GOING TO BE LEFT BEHIND BECAUSE THE COMMERCIAL MARKET WILL HELP US GET THERE. THAT'S NOT AN ABSOLUTE GIVEN BUT I THINK IT PROBABLY WILL FROM WHAT WE'VE SEEN IN THE PAST. AN EXCITING TIME AND HOPEFULLY THIS CONTINUED PARTNERSHIP WILL LEAD US TO BRING THESE GENE THERAPIES TO PATIENTS SOONER AND MAKE A DIFFERENCE IN PEOPLE'S LIVES. SO I WILL STOP THERE. THANK YOU. >> OVER TO CHRISTINE NOW. >> THANK YOU SO MUCH TO ALL OF OUR SPEAKERS. YOUR DEDICATION TO THE PURSUIT OF TREATMENTS AND CURES FOR RARE DISEASE COMMUNITY IS VERY MUCH APPRECIATED. IT'S CLEAR THE MOST IMPORTANT THING IS TREATMENT AND CURES TO TREAT MANY DISEASES AT A TIME WILL REQUIRE NCATS TO WORK IN PARTNERSHIP WITH RESEARCHERS, PATIENTS, ADVOCACY ORGANIZATIONS AND INDUSTRY. AND NCATS' ABILITY TO SERVE AS A CONVENER WILL BE INSTRUMENTAL MOVING FORWARD AND THE COLLABORATION IS EVIDENCE OF HOW CLOSELY NIH WORKS WITH REGULATORY PARTNERS. AS A CAREGIVER AND HEALTH POLICY PROFESSIONAL I HOPE WE CAN ENSURE ACCESS TO TREATMENTS AND CURES AS THEY ENTER THE MARKET. ONE OF THE GREATEST BARRIERS TO DISCOVERY AND DEVELOPMENT OF TREATMENT IS DIAGNOSIS AND UNDERSTANDING OF THE NATURAL [NO AUDIO] AND MY DAUGHTER'S RARE DISORDER IS PART OF THE NEW REGISTRY PROGRAM. PROGRAM DEVELOPED AT NO COST TO PATIENT ORGANIZATIONS YET STILL ENSURING PATIENT OWNERSHIP OF THE DATA ARE CRITICAL TO UNDERSTANDING THE NATURAL HISTORY OF THE DISEASE TO PAVE THE WAY FORWARD FOR TREATMENTS AND CURES. OUR NATION LEARNED A GREAT DEAL FROM THE COVID RESPONSE AND I LOOK FORWARD TO LEARNING MORE HOW LESSONS FROM THE COVID RESPONSE CAN BE TAKEN AND APPLIED TO RARE DISEASES. I SEE HOPE IN NCATS' WORK TO THE WORK IN THIS SPACE AND LOOK FORWARD TO WATCHING CONSORTIUM PARTNERS TAKE GENE EDITING AND APPLY THEM IN A SCALABLE WAY. THANK YOU ALL FOR TAKING THE TIME TO BE WITH US. WE NOW GO INTO A BREAK AND RECONVENE AT 3:40 P.M. FOR THE NEXT SESSION. THANK YOU. >> WELCOME BACK TO NCATS CELEBRATION AND THIS IS THE FUTURE OF CLINICAL RESEARCH THOUGH IT SOUNDS LIKE A NEW YORK CITY NIGHTCLUB ON THE LOWER EAST SIDE IT'S A SESSION THAT WE'LL TALK ABOUT WITH A VERY ECLECTIC PANEL OF THE FUTURE OF WHAT WE CAN ANTICIPATE IN THE AREA OF CLINICAL RESEARCH. BEFORE WE GET TO THAT I WANTED TO GIVE BACKGROUND CON TEST. THE SPEAKERS WILL BE ALL FROM THE CTSA PROGRAM. I'M MIKE KURILLA THE DIRECTOR OF THE DIVISION OF CLINICAL INNOVATION THE EXTRAMURAL FUNDING ARM AND HOME AND RESPONSIBILITY FOR THE OVERSIGHT AND MANAGEMENT OF THE CTSA OR CLINICAL AND TRANSLATIONAL SCIENCE AWARD PROGRAM. THE PROGRAM INTERESTINGLY ENOUGH IS MUCH OLDER THAN NCATS GOES BACK TO 2006 WHEN IT WAS OVER SEEN BY THE NATIONAL CENTER FOR RESEARCH RESOURCES AND IS WAS THE BRAIN CHILD OF THE PRIOR NIH DIRECTOR ELIAS SURHUNI IN HIS REINVENTING THE CLINICAL ENTERPRISE. THERE WAS A RECOGNITION THAT AS WITH EVOLUTION IN TECHNOLOGY NOT ONLY WHAT WE STUDY IN SCIENCE BUT THE WAY WITH DO SCIENCE AND THE WAY WE CONDUCT MEDICAL RESEARCH SHOULD IN FACT CHANGE AND EVOLVE OVER TIME AND THE CTSA PROGRAM WAS ONE APPROACH TO TRYING TO RESPOND TO THOSE CHANGING CONDITIONS SO THE CTSA PROGRAM WAS BORN WITH THE FOCUS ON MUCH MORE ATTENTION TO THE AREA OF CLINICAL RESEARCH IN PARTICULAR THE TRAINING OF INVESTIGATORS IN THE FIELD WITH A FOCUS ON TRANSLATIONAL SCIENCE SO THAT ALL OF THE WONDERFUL SCIENCE AND BREAKTHROUGH DISCOVERIES NIH WAS MAKE COULD MORE RAPIDLY AND EFFICIENTLY BE TRANSLATED FROM SCIENCE INTO HEALTH AND HAVE DIRECT APPLICABILITY TOWARDS THE OVERALL PUBLIC HEALTH SECTOR. SO THE PROGRAM MATURED FROM THE GENERAL CLINICAL RESEARCH CENTERS AND BY THE TIME IT WAS TRANSFERRED TO NCATS IT HAS MATURED AND GROWN TO ITS PRESENT SIZE. WITH THE POTENTIAL FOR THE FULL CONSORTIUM OF THE APPROXIMATELY 60 ACADEMIC MEDICAL CENTERS FORMING THE CTSA PROGRAM THE PROGRAM BEGAN TO FOCUS ON SOME CONSORTIUM WIDE ACTIVITIES AND WE'VE SEEN TREMENDOUS EFFORTS IN TERMS OF THE DEVELOPMENT AND INFORMATION OF INFORMATICS AND FOCUSSING ON HIGH LEVEL ISSUES AND CONCERNS IN TERMS OF DATA INTEROPERABILITY ESPECIALLY IN THE REALM OF ELECTRONIC HEALTH RECORDS AS MATURED IN THE COVID ERA TO ENABLE AND PRODUCE N3C THE NATIONAL COHORT COLLABORATIVE. THE LARGEST DATABASE OF EHR RECORDS CONCERNING COVID PATIENTS CURRENTLY AT OVER 3 MILLION. WE'VE ALSO SEEN WONDERFUL DEVELOPMENTS IN TERMS OF FOR EXAMPLE SMART IRB WHICH HAS ENABLED RELIANCE AGREEMENTS FOR MULTI-CENTERED SITES TO PROCEED FROM TRADITIONALLY WHAT TOOK MONTHS OF NEGOTIATIONS DOWN TO WEEKS OR DAYS OR IN THE CASE OF ONE COVID TRIL TOOK 16 MINUTES TO INITIATE A MULTI-CENTERED TRIAL. THE CTSA PROGRAM FOCUSSED ON ACTIVITIES TO PROMOTE ACTIVITY AND REACH IN TERMS OF THE ABILITY TO CLINICAL RESEARCH TO UNDERTAKE CRITICAL AND NECESSARY ACTIVITIES. SO OUR PANEL TODAY WHICH CONSISTS OF WILBUR LAM FROM EMRY AND GEORGIA INSTITUTE AND TECHNOLOGY AND CHILDREN'S HEALTH CARE AND THE PROFESSOR AND THE W.B. BOWERS IN PEDIATRICS AND BIOMEDICAL ENGINEERING AND THEN WE HAVE ALEJANDRA HURTADO DE MENDOZA AND OLVEEN CARRAQUILLO AND ERIC TOPOL OF THE FOUNDER OF THE SCRIPPS TRANSLATIONAL INSTITUTE TO PROVIDE BROAD CLOSING REMARKS. UNFORTUNATELY WE'VE BEEN HAVING SOME TECHNICAL DIFFICULTIES WITH WILBUR SO WE'LL MOVE DIRECTLY TO ALEJANDRA SO TAKE IT AWAY. >> THANK YOU FOR THAT INTRODUCTION. IT'S A PLEASURE TO BE HERE TO PRESENT MY WORK AND WE HAVE LOOKED AT DEVELOPING INTERVENTIONS THROUGH DISPARITIES IN THE USE OF SERVICES FOR MINORITIES AT RISK OF OVARIAN AND BREAST CANCER. WE KNOW ADVANCES IN PRECISION MEDICINE AND LOOKING AT GENOMIC MEDICINE AND FOCUSSING ON HEALTH EQUITY. MOST ECONOMIC STUDIES HAVE FOCUSSED ON NON-HISPANIC WHITE POPULATIONS FROM ACADEMIC MEDICAL CENTERS AND IMPLEMENTATION SCIENCE HAS BARELY BEEN USED UP THIS FIELD. THE FUTURE LINES OF RESEARCH TO MAKE SURE PRECISION MEDICINE CAN RENEW DISPARITIES INCLUDE INCREASING REPRESENTATION OF MINORITIES IN RESEARCH, ENGAGING COMMUNITIES TO DEVELOP CULTURALLY RELEVANT MATERIALS THAT INCREASE THE ACCESS TO TESTING BY REDUCING BARRIERS SUCH AS REFERRING OR LANGUAGE BARRIERS AND USING IMPLEMENTATION SCIENCE TO EXAMINE WAYS OF DELIVERING GENETIC SERVICES IB COMMUNITY SETTINGS AND CLINICS. NOW I'M GOING TO GIVE EXAMPLES OF MY WORK IN THIS LINE OF RECOMMENDATIONS. THIS WAS A VIDEO BECAUSE WE HAVE WE INTERVIEWED AT-RISK LA TINA WOMEN AND DEVELOPED THE VIDEO AND WE ALSO PARTNERED WITH COMMUNITY BASED ORGANIZATIONS. WE ENDED UP WITH THIS VIDEO OF 18 MINUTES AND HAVE PROMISING FINDINGS IN THAT WOMEN IN A SINGLE ARM TRIAL CLINICED INCREA CLINICS AND THIS WAS PROMISING FINDINGS IN A SMALL PILOT. I RECENTLY GOT AN RO1 GRANT LAST YEAR TO TEST THIS INVENTION TO A RANDOMIZED CONTROL TRIAL WITH 300 WOMEN. IN ADDITION TO TESTING THE EF EFFICACY AND MECHANISMS THERE'S AN IMPLEMENTATION SCIENCE AIM BECAUSE A BARRIER FOR WOMEN PARTICIPATING IS THEY RECEIVE CARE IN CLINICS AND THEY DON'T HAVE AVAILABLE RESOURCES TO SCREEN WOMEN AND REFER WOMEN IN SERVICES. IN THIS ARM WE'RE INTEGRATING A SHORT VALIDATED SCREENING AND LOOKING THET DATA FOR IMPLEMENTING THE SCREEN AND HOW TO MAKE THIS SUSTAINABLE. WE'RE EXAMINING HOW TO INTEGRATE THE VIDEO AND SCREENING SYSTEM AND PROCESS SO THE SYSTEMS CAN BE SUSTAINABLE AND STAY IN PLACE. AND THERE'S A CAREER AWARD AND WE ADAPTED AN INTERVENTION FOR AT RISK LATINA WOMEN TO IN ADDITION TO KNOWLEDGE WOMEN HAVE LOGISTIC BARRIERS SUCH AS TRANSPORTATION. BY DOING GENETIC COUNSELLING INSTEAD OF IN PERSON COUNSELLING WE CAN ADDRESS THE BARRIERS INCLUDING COST BARRIERS AS WELL. ONE OF THE OTHER MAIN BARRIERS IS LANGUAGE. THERE'S FEW SPANISH SPEAKING GENETIC COUNSELORS IN THE U.S. AND WE CAN INCREASE THE ACCESS TO THE SPANISH SPEAKING COUNSELORS IN THE U.S. WE ADAPTED AN INTERVENTION. THIS IS THE REGIONAL BOOKLET THAT GOES WITH THE COUNSELLING SESSION AND HOW WE ADAPTED IT. AND LOOK AT VALUES AND MAKE SURE THEY'RE EMBEDDED IN THE INTERVENTION. WE HIRED ADMINISTRATORS TO MAKE SURE WE HAVE IMAGES THAT WE COULD FEEL WAS REPRESENTED. IN ADDITION TO THAT, WE HAD TO BE FLEXIBLE BECAUSE IN ORDER TO HAVE PARTICIPANTS RECEIVING TESTING WITHOUT HAVING GENETIC COUNSELLING. THAT WAS PROBLEMATIC BECAUSE A LOT OF WOMEN DOESN'T UNDERSTAND THE TEST RESULTS OR IMPLICATION FOR FAMILY MEMBERS AND HAD TO INCLUDE WOMEN WHO WERE TESTING SO WE INCLUDED THE POPULATION BECAUSE IT WAS IMPORTANT FOR THEM TO UNDERSTAND THE TEST RESULTS. ANOTHER THING WE DISCOVERED THROUGH THE PROTOCOL IS THOUGH OUR INVENTION WAS SUCCESSFUL IN INCREASING PARTICIPATION IN COUNSELLING IT WAS SIGNIFICANTLY INCREASED ED TO USUAL CARE ARM AND THE RATES OF TESTING WAS LOW AND WE KNOW WITH THE USE OF TELEMEDICINE THE GENETIC TESTING IS LOWER WHEN A PARTICIPANT YEB ENCOUNTERED COUNSELLING. WE REALIZED WOMEN WERE HAVING TROUBLE UNDERSTANDING THE INSTRUCTIONS IN HOW TO COMPLETE THE SALIVA SAMPLE AND SOMETIMES IT'S HARD IT UNDERSTAND. WE HAD ONE OF OUR COMMUNITY PARTNERS SHOOT A VIDEO ON HOW TO COLLECT SALIVA SAMPLE IN A CULTURALLY ADAPTED WAY. IT WAS HELPFUL HOWEVER, SOME WOMEN STILL HAD BARRIERS IN TERMS OF LOGISTICS THAT NEED TO BE ADDRESSED AND ONE FUTURE LINE OF RESEARCH IS TO UNDERSTAND FACILITATORS AND TRY TO UNDERSTAND THE STRATEGIES TO IMPROVE THE BIO MEDICINE DATA COLLECTION FOR TELEMEDICINE. I THINK IT WILL BE KEY FOR THIS FIELD AND FOR OTHER FIELDS. LASTLY I'D LIKE TO SHARE THIS IN PARTNERSHIP WITH OUR COMMUNITY BASED ORGANIZATIONS WE KNOW HOW THE PANDEMIC HAS IMPACTED MINORITIES SO IN THIS GRAND WE INTENT TO EVALUATE THE WELL BEING OF AFRICAN AMERICAN AND LATINA BREAST CANCER SURVIVORS AND DID SERVICES WITH 79 BREAST CANCER SURVIVORS AND WITH ALL THE INFORMATION WE LAUNCHED THIS A COUPLE WEEKS AGO AND WE USED ALL THE INFORMATION WE LEARNED IN OUR INFORMATIVE WORKS. ONE IS WOMEN DIDN'T TRUST THE SOURCES OF INFORMATION FOR COVID-19 AND WE BROUGHT THE VOICES FROM THE COMMUNITY TO THE WEBSITE AND INCLUDED QUOTES FROM THE INTERVIEWS AND INCLUDED OUR COMMUNITY PARTNERS AS ROLE MODELS YOU SEE IMAGES OF WOMEN AND TESTIMONIALS WHY IT WAS IMPORTANT TO GET THE VACCINE AND LEARNED WOMEN DIDN'T HAVE INFORMATION ABOUT COVID-19 AND THE VACCINES FOR BREAST CANCER SURVIVORS SPECIFICALLY. WE ALSO ADDED THAT INFORMATION. THE GOAL OF THE WEBSITE IS TO EMPOWER THE WOMEN WITH THIS INFORMATION IN A WAY THAT'S CULTURALLY RELEVANT. I WANTED TO HIGHLIGHT THE IMPORTANCE OF WHEN WE THINK ABOUT PRE VISION PED SIN AND MAKE SURE PRECISION MEDICINE IS BENEFICIAL TO ALL POPULATIONS WE NEED TO MAKE SURE WE INCREASE REPRESENTATION OF MINORITIES IN RESEARCH, MAKING SURE THE INTERVENTIONS DEVELOP A TARGET FOR MINORITY POPULATIONS FOR FACILITATORS IN ORDER TO DO THAT IT'S KEY TO ENGAGE COMMUNITY BASED ORGANIZATIONS INCLUDING PATIENT ADVOCATES AND ALL THE PHASES OF RESEARCH BECAUSE THEY'RE THE ONES THAT ARE THE TRUSTED SOURCES OF THE COMMUNITY. IT'S VERY IMPORTANT TO BE FLEXIBLE. I THINK I SHARED EXAMPLES HOW WE HAVE THE PROTOCOLS WITH THE CRITERIA AND INTERVENTION MATERIALS TO BETTER ADDRESS THE NEEDS OF THE POPULATION AND BETTER MAKE THEM BETTER WITH CLINICAL PRACTICE AND I WANT TO STRESS THE IMPORTANCE OF IMPLEMENTATION SCIENCE AND CONSIDER OUTCOMES TO THE INTERVENTIONS IMPLEMENTED IN ACTUAL CLINICAL PRACTICE. THANK YOU VERY MUCH. THANK YOU JONI FOR INVITING ME TO PRESENT. UNTIL A WEEK AGO I WAS CHIEF OF PRIMARY CARE AT THE UNIVERSITY OF MIAMI WHERE I HAD RUN A LARGE HEALTH CARE DELIVERY AND RUNNING SEVERAL RESEARCH PROGRAMS. I WAS RECENTLY SERVE AS MPI FOR OUR CO-DIRECTOR AND AMONG MY MANY ROLES I ALSO AND BASICALLY I CROSSED OFF TWO OF FRANCIS COLLINS' ACHEEIEVEMENTS AND I JUST NEED TO FIGURE OUT HOW TO GET ON THE BRAIN INITIATIVE. THE PANDEMIC HAS SHOWED US NEW WAYS TO WORK TOGETHER AND IN THE SPACE I WORK WITH COMMUNITY ENGAGEMENT COVID MORE THAN ANYTHING ELSE HAS SHOWN A CRITICAL ROLE THAT COMMUNITY HAS BEEN PART OF THE CONTINUUM. I STILL REMEMBER 10, 10 YEARS AGO WHEN THERE WAS A ROLE OF THE SPECTRUM AND I THINK JUST LIKE WITH BASIC SCIENCE, COVID ALSO MADE CLEAR THE IMPORTANCE OF ALSO COMMUNITY ENGAGED RESEARCH IN SOLVING THE VEXING PROBLEMS OF OUR SOCIETY. TO ME SEEMS KIND OF SILLY. THE BENEFACTOR OF OUR WORK AS A COMMUNITY WE NEED TO WORK WITH COMMUNITY THE MODEL WE USE IS AT THE BRIDGE BETWEEN THE COMMUNITY AND HEALTH CARE THROUGH RESEARCH. WE'VE BEEN INVOLVED IN TESTING COMMUNITY HEALTH WORKER INVENTIONS IN CANCER, DIABETES, HEART DISEASE AND HIV AND CREATING TRAINING CURRICULUM AND HAVE SHOWN THEY WORK. THEY WERE EFFECTIVE IN REACHING THE POPULATION AND GET THEM TO OUR STUDY AND THE SOLUTIONS THEY HELP COME UP WITH. ARE THEY ATTUNED TO THE NUANCES OF A TARGET COMMUNITY AND HELP IN APPROACHES FOR THE COMMUNITY. AND THERE'S APPROACHES WE LOOKED AT DO QUITE WELL IN TRANSLATIONAL RESEARCH. ANOTHER PERFECT EXAMPLE ALSO THAT NCATS HAS BEEN INVOLVED WITH AND THESE ARE STATEWIDE COALITIONS ADDRESSING AT MISINFORMATION AND DIVERSITY AND VACCINE UPTAKE. AND IT'S TAKEN A LEAD ROLE IN THE INITIATIVES. AND DISRUPTIVE MISINFORMATION AND PEOPLE ON THE EDGE DUE TO RACIAL STRIFE, POLICE BRUTALITY, POLITICS AND PEOPLE HAVE PREYED ON THOSE THINGS TO SPREAD DISINFORMATION. AND HOW DO YOU BATTLE THIS AND WE KNEW WE HAD TO ENGAGE THE COMMUNITY AND WE HAVE SEEN MUCH SUCCESS AND OTHER PARTS OF THE COMMUNITY ENGAGE THE PARTNERING WITH COMMUNITIES TO ADDRESS THE MISINFORMATION TARGETING THEIR COMMUNITIES. AND I HEAR ALL THE GREAT EXAMPLES OF PEOPLE DOING IT SLIGHTLY IN EACH STATE WORKING WITH DIFFERENT SOLUTION. I SAW VIDEOS FROM THE PUERTO RICO TEAM AND WHAT THEY'RE DOING THERE AND WE MUST ALL ADDRESS THE COMMUNITY AND MANY WERE AFRAID MINORITIES WOULDN'T PARTICIPATE IN THE STUDIES AND THROUGH THE UNIVERSITY OF MIAMI WE HAD OVER 60% MINORITY PARTICIPATION IN THE VACCINE TRIALS. THE ALL OF US PROGRAM WE RUN IT WITH EMORY AND WE ACHIEVED OVER 70% DIVERSITY BY HAVING THE COMMUNITY-ENGAGED APPROACHES WORKING AND TESTING THE LAST DECADE. AND WHEN YOU TALK ABOUT OTHER PROGRAMS LIKE THE CTSI THEY ALL ACHIEVE SIMILAR SUCCESS BECAUSE OF THE COMMUNITY ENGAGED APPROACHES AT THE PROGRAMS BEGINNING TO FORMULATE. AND WE RECENTLY HIGHLIGHTED A PAPER ON COVID COMMUNITY ENGAGEMENT DURING COVID AND ONE OF THE THINGS ACROSS SEVERAL CTSAs WITH US THE ROLE THE COMMUNITY ENGAGEMENT CORPS PLAYED AND YOU NEED THEM IN THE TIME OF CRISIS AND AUTHENTIC STAKEHOLDER ENGAGE THE ARE PARAMOUNT IN WORKING WITH THE PARTNERS HOWEVER, THE CHALLENGE IS THE INFRASTRUCTURE FOR SUPPORTING LONG TERM ENGAGEMENT AND WE LEVERAGE THE STRUCTURE TO ADDRESS THE PANDEMIC AND THAT'S KEY. ANOTHER THING COVID TAUGHT US WERE NEW ENGAGEMENT METHODS. A LOT OF THEM WERE VIRTUAL. WE'RE WORKING ON THINGS LIKE ELECTRONIC RESEARCH REGISTRIES THROUGH THE ELECTRONIC MEDICAL RECORDS FOR MINORITY POPULATIONS THROUGH THE STUDY AND WE TALKED ABOUT E-CONSENT AND VIRTUAL CONSENT AND WE FOUND OUT DURING COVID THE THINGS THAT WERE WORKING IT WAS POSSIBLE TO LEVERAGE TECHNOLOGY AND VIDEO CONFERENCING TO SUPPORT REMOTE ENGAGE THE AND HELPED MAINTAIN OUR PROJECT AND MANY OTHER STUDIES WE FOUND WORK AROUNDS AND WE SAW GROUPS BEING LEFT OUT AND NEED TO MAKE SURE ALL OF US HAS BEEN AT THE FOREFRONT BUT SEE FOR SOME PEOPLE THEY STILL NEED THE HAND HOLDING AND STILL NEED TO BRING THEM UP AND NEED A PERSON TO PERSON APPROACH. AT THE END OF THE DAY FOR SOME PEOPLE THESE DIGITAL APPROACHES WE PIONEERED WILL NOT WORK SO WE NEED TO KEEP THOSE GROUPS. TO THE VIRTUAL ENGAGEMENT APPROACH DOES NOT ENGAGE FACE TO FACE AND THE VIRTUAL ENCOUNTERS ARE GREAT BUT MISS THE PERSON INTERACTIONS AS WELL. AND WE LEARN ABOUT TRUST WORTHINESS. WE'VE HAD A LOT OF TALKS ABOUT THIS AND WE HEARD ABOUT HORRIBLE ABUSES IN THE PAST, TUSKEGEE, EVERY COMMUNITY HAS A LIST OF ABUSES AND STILL NEED TO RECOGNIZE THESE THINGS ARE STILL GOING ON AND MAYBE NOT AS EGREGIOUS BUT YOU SEE EXAMPLES OF HOW IT'S ONGOING STILL AND NEED TO FOCUS ON THE TRUSTWORTHINESS WITH COMMUNITY PARTNERS. LONG-TERM TRUST WE NEED THE FUNDING TREME TO MAKE THIS HAPPEN. THE COMMUNITY ALSO WANDS TO SEE YOU AS A VISIBLE VOICE AND IT'S NOT JUST ABOUT THE IT'S ABOUT THE HEALTH ISSUE OR WHATEVER THE CONCERN IS AND WE ALSO NEED TO SUPPORT THE COMMUNITIES IN APPROACHES THEY TOLD YOU WILL WORK LIKE COMMUNITY HEALTH WORKERS. AND THE CTAs HAVE BEEN FRONT AND SFRT AND MANY COMMUNITY PARTNERS CAN'T FLOAT SIX, SEVEN MONTHS AND THE APPROACHES THE N CAT HAS HELPED SPEED THIS UP. NONETHELESS WE STILL HAVE WORK TO DO. ACADEMIC INSTITUTIONS STILL TO KNOW WHAT STILL EXISTS AND HOW HEALTH CARE IS DELIVERS BY COMMUNITY PARTNERS IS CRITICALLY IMPORTANT AND THE EMERGENCY ROOM AND WHAT KIND OF PATIENTS THEY SERVE AND WE NEED TO FOCUS ON THOSE THINGS. MY WORK IS A BAND-AID AND WE REALIZE THERE'S SOCIAL DETERMINATES OF HEALTH. LACK OF HEALTH INSURANCE, POOR ACCESS AND POVERTY, NEIGHBORHOODS, AND POLITICAL EMPOWERMENT ARE TE KEY DRIVERS AND I THINK WE NEED TO THINK MORE CREATIVELY. MANY HAVE DEVELOPED CLEAR EXAMPLES OF THE BAND-AIDS BUT WE NEED TO ADDRESS THE FUNDAMENTAL DRIVERS AND NEED TO ADDRESS THESE AS THE KEY DRIVERS. WE NEED TO TALK ABOUT SPEEDING UP DISCOVERIES AND GETTING THEM TO PATIENTS FASTER BUT AT THE END OF THE DAY WE'LL TALK ABOUT THE POPULATIONS AND NEED TO THINK MORE STRATEGICALLY ABOUT ADDRESSING THE SOCIAL DETERMINATES OF HEALTH AS A KEY DRIVER. LAST THE LAST POINT HAS BEEN THE MOVEMENT AND I KNOW MANY OF US DON'T LIKE TO TALK ABOUT IT BUT WHEN I TALK ABOUT REACHING ALL SEGMENTS OF THE COMMUNITY WE WANT TO REACH EVERYONE. WE'VE SEEN COVID DRAMATIC UPTAKE BY PARTY AFFILIATION AND NEED TO BATTLE BACK TO MAKE SURE THE TRANSLATIONAL DISCOVERIES REACH EVERYBODY WHETHER THEY'RE MINORITIES OR BELIEVE IN SCIENCE OR NOT. AND WITH THAT I WILL STOP AND HAND IT BACK TO MICHAEL I THINK. >> IT'S NOT A VIRTUAL MEETING UNLESS YOU HAVE TECHNICAL GLITCHES ALONG THE WAY BUT IT LOOKS LIKE WE HAVE WILBUR BACK. IS EVERYTHING IS OKAY TAKE IT AWAY. >> HOPEFULLY YOU CAN HEAR ME. >> SO THANK YOU SO MUCH FOR HAVING ME. MY NAME IS WILBUR LAM. I'M A PEDIATRIC HEMATOLOGIST ONCOLOGIST AT EMORY UNIVERSITY IN CHILDREN'S ATLANTA AND PROFESSOR OF BIOMEDICAL ENGINEERING AND EMORY AND GEORGIA TECH AND DIRECTOR OF THE INNOVATION CATALYST PROGRAM WITH RPSC BASED AT EMORY UNIVERSITY, GEORGIA TECH AND MOORE HOUSE AND UGA. WHAT I'LL TALK TO ABOUT HOPEFULLY AND WHAT YOU SEE IS AN EXAMPLE OF AN APP WE HELPED OTHER CLINICIAN COLLEAGUES BUILD AND PEDIATRIC CARDIOLOGIST THAT DOES CONGENITAL HEART DISEASE SCREENING. A COUPLE DISCLOSURES. I STARTED A FEW START-UP COMPANIES FROM MY OWN RESEARCH WORK. TWO I'LL TALK ABOUT AND ONE RECENTLY ACQUIRED AND ONE STILL ONGOING. AND TALK ABOUT MY OWN ADVENTURES IN DIAGNOSTIC TESTING. THIS STARTED BACK WHEN I WAS IN GRADUATE SCHOOL. I WAS A PA FOR MY Ph.D ADVISER AND TALKING OPTICS COURSE. ONE CHALLENGE WE OPPOSED THE UNDER GRADS WAS COULD WE TAKE MOBILE PHONES AND USE OFF THE SHELF PARTS AND MAKE THOSE MOBILE PHONES INTO MICROSCOPES. AND LONG STORY SHORT AFTER A COUPLE YEARS IT WORKED. AND DO WE DO TUBERCULOSIS AND WHAT HAPPENED WAS MY OLDER CHILD AT THE TIME WAS JUST A TODDLER AND KEPT GETTING EAR INFECTIONS AND LUCKILY I WAS A PEDIATRICIAN AND NEWLY BOARDED PEDIATRICIAN AND LOOK IN HIS EARS AND SAY INFECTION, NO INFECTION AND ULTIMATELY ENDED UP HAVING CHRONIC EAR INFECTIONS AND MY WIFE WHO IS NOT IN MEDICINE SAID WHAT HAPPENS WITH OTHER FAMILIES? I SAID, WELL, IF YOU HAVE A KID WITH EAR INFECTION YOU BRING THEM TO THE EMERGENCY ROOM AND THE FIRST AND ONLY TIME SHE SAID THIS, SHE SAID I'M GLAD I'M MARRIED TO YOU. WHAT THAT REALLY SHOWED IT GAVE US OUR CLINICAL APPLICATION FOR POTENTIAL CELL PHONE MICROSCOPE AND THEN 2007 HAPPENED AND THEN WITH THE AT ADVENT OF THE CLINICAL APPLICATION AND A GADGET THAT COULD BE WORTHWHILE AND USED FOR EAR INFECTIONS. AND WE FORMED A COMPANY CALLED CELL SCOPE AND ERIC WAS BE NICE TO JOIN OUR TEAM BECAUSE HE WAS GOING ON THE COLBERT REPORT AND IF YOU ASK MY MOM EVEN TODAY WHAT I DO, SHE HAS NO IDEA BUT THE ONE THING SHE IS ABLE TO ARTICULATE I CO-INVENTED A FAMOUS DOCTOR PUT IN THE EARS OF A RUDE MAN ON TV AND THIS IS WHEN HE WAS STILL IN CHARACTER. AND THIS SYSTEM WILL BE USED WITH A HEALTH CARE SUITE. ANOTHER PROJECT OUR LABORATORY WORKED ON WAS FOCUSSED ON ANEMIA IN ASSESSING HEMOGLOBIN LEVELS. AS A HEMATOLOGIST I SEE A LOT OF PATIENTS WITH ANEMIA AND ONE THING WE THOUGHT ABOUT WAS WHETHER OR NOT WE COULD QUANTIFY POWER, PALENESS ON DIFFERENT BODY PARTS. THE IDEA WE HAD FOR A WHILE AND I WAS LUCKY TO BE PARTNERS WITH ONE OF MY GRADUATE STUDENTS AT THE TIME WHO IS NOT ONLY A BRILLIANT COMPUTER CODER BUT ALSO HAPPENS TO HAVE A GENETIC BLOOD DISORDER AND THAT ALOUD US TO GET OUR FIRST DATA SET AND IT GOES FROM NOT ANEMIC TO ANEMIC AND WITH THIS INITIAL DATA SET WE WERE ABLE TO ESTABLISH FEASIBILITY AND SAY WE HAVE SOMETHING HERE. AND ULTIMATELY WE DEVELOPED THIS ROBUST ALGORITHM TO PREDICT AND ESTIMATE HEMOGLOBIN LEVELS BASED ON TAKING PICTURES OF A FINGER NAIL SELFIE. AND IN 2006 WE LAUNCHED OUR FIRST APP ON GOOGLE PLAY AND THE GOOGLE APP STORE. ANYONE CAN DOWNLOAD IT AND ESTIMATE THEIR OWN HEMOGLOBIN LEVELS WITH THEIR FINGER NAILS. AGAIN, I GAVE MY DISCLOSURE AND MOBILE PHONES ARE REALLY USEFUL. GOING BACK TO MY TEAM AT THE CTSA WE SCRATCHED OUR HEADS AND SAID WHAT CAN WE DO HERE? AND WOULD WE BRING MULTIPLE APPS AND DEMOCRATIZE AND BUILD APPS FOR OUR FACULTY AND STAFF AND STUDENTS TO CONDUCT CLINICAL RESEARCH AND WE SAW A LOT OF GREAT OPPORTUNITIES HERE AND WE SAW EVEN SIMPLE APPS HAVE CLINICAL VALUE WHEN IT COMES TO RESEARCH THAT DON'T NEED BIG DATA AND ACCESS TO EMR BECAUSE APPS ARE SO UBIQUITOUS AND THERE'S THE TECHNICAL ISSUES, WHO WILL BUILD THE SYSTEM? AND SUBMITTING THE APP TO GOOGLE IS LIKE SUBMITTING A PAPER TO A JOURNAL WITH A REALLY CAPRICIOUS EDITOR AND THE BACK END INFRASTRUCTURE WHO WILL RUN THE APP IN THE BACKGROUND AND WHERE WILL THE DATA BE STORED AND DATA AND ANN SOFTWARE REQUIRES A BIT OF MAINTENANCE. WE SAID WE HAVE CHALLENGES AND OPPORTUNITIES HERE. WE'LL MAKE A CORE SERVICE WITHIN OUR CTSA WE CALL THE APP HATCHERY. WHAT WE LEARNED IS WE NEED TO TAKE A USER CENTERED DESIGN APPROACH AND APPS ARE EVERYWHERE AND THE OPPORTUNITY AND SOMETIMES PART OF THE PROBLEM AND SOMETIMES CLINICIANS COME IN WITH PRECONCEIVED NOTIONS OF WHAT THEY THINK THE APP SHOULD DO WHEN SOMETIMES THEY UNDERSTAND APPRECIATE THE IDEA WITH THE USER OR PATIENT POPULATION IT'S NOT EXACTLY HOW THEY WOULD ENGAGE IN THE ACT TO ADDRESS THE CLINIC PROBLEM OR ASK THE CLINICAL QUESTION WE WANT TO. WHAT WE DO IN THE APP HATCHERY WE BACK AWAY FROM THE FEATURES AND SAY HOLD ON CLINICIAN WE KNOW HAVE YOU IDEAS BUT LET'S SOLVE SPECIFIC CLINIC PROBLEM OR HELP ANSWER YOUR SPECIFIC CLINICAL RESEARCH QUESTION IN THE WAY THE USER WILL KNOW EXACTLY HOW TO ENGAGE AND ACHIEVE YOUR GOAL AND TRY TO LIVE IN THE SPACE WHERE IT'S A COMBINATION OF THE RIGHT TECHNOLOGY AND RIGHT CLINIC NEED AND RIGHT WAY TO ENGAGE THE USERS. WE TALKED TO THE DIFFERENT STAKEHOLDERS AND PATIENTS THAT ARE SIMPLE APZ WILL INVOLVE. HERE'S AN EXAMPLE, THIS IS A PROJECT WE CALL HOMETOWN. THIS IS GEARED TOWARDS PATIENTS WITH SOME GENETIC CANCER PREDISPOSITION AND OUR COLLEAGUES SAID THESE PATIENTS ARE AT RISK FOR CANCERS BUT HAVE SO MUCH GONE ON WITH THEIR LIVES THEY'RE OFTEN DEALING WITH MULTIPLE CANCERS AND END UP MISSING MANY OF THEIR FOLLOW-UP APPOINTMENTS. COULD WE MAKE A FAIRLY SIMPLE APP TO HELP THEM MAKE THEIR APPOINTMENT AND THEREFORE POSSIBLY IMPROVE CLINICAL OUTCOMES AND METRICS AND WHAT WE LEARNED WITH THE USER RESEARCH IS THESE S ARE NOT IN ISOLATION. THEY'RE NOT ALONE. THEY'RE FAMILIES. WE FIGURED OUT THERE'S MULTIPLE STAKEHOLDERS AND TOOK THE NOTION A COLLEAGUE HAD AND BLEW IT UP SO THERE'S MORE USERS THAT WERE BEING SERVED AND NOW WE'RE AT THE PROTOTYPE STAGE WHERE WE'RE STARTING TO HAVE FAMILIES WITH GENETIC CANCER PREDISPOSITION STARTING TO USE THIS TYPE OF DEVICE AND THIS IS ONE EXAMPLE. WE HAVE ABOUT 20 OTHER PROJECTS WE'RE WORKING WITH IN OUR CTSA IN MATERIALS OF MAKE SIMPLE APPS FOR OUR CLINICIAN COLLEAGUES. AND WE NEED SOMEONE WHO IS THE INVENTORS OF THE FIRST APP I TOLD YOU ABOUT WITH AN ESTIMATED HEMOGLOBIN AND IS NOW SERVING AS THE TECHNICAL LEAD WITHIN THE APP HATCHERY. SOMEONE WITH USER DESIGN EXPERIENCE AND OUR TEAM WHO PUTS IT ALL TOGETHER IN MATERIALS OFF THE PRODUCT STRATEGIES AND HAVE BRILLIANT DEVELOPERS ON OUR TEAM. AND WITH THAT, THANK YOU SO MUCH. >> HELLO. I'M SO GLAD TO JOIN. FIRST, CONGRATULATIONS TO NCATS ON THE 10 YEAR ANNIVERSARY. I'VE THAT'D PLEASURE ON SERVING ON THE NCATS COUNCIL A COUPLE YEARS AND SAW THE REMARKABLE PEOPLE BEHIND THE SCENES AT NIH AND THE GREAT PRIVILEGE OF SERVING AS A CTSA DIRECTOR SINCE 2008 AT SCRIPPS RESEARCH. THE PROGRAM HAVE BEEN TRANSFORMATIVE AND TRYING TO GET TO THE NEXT CHAPTER. AND IF I DON'T KEEP GETTING PROMPTS HERE FROM THE LAB WHICH I HAVE TO SAY IS MORE AWKWARD THAN THE ZOOM PLATFORM, OKAY. I WANT TO ALSO THANK YOU FOR THE MEMORY OF THE STEPHEN COLBERT EPISODE AND NOW I WANT TO TALK ABOUT ASSEMBLY OF THE PARTS. WITH THE PANDEMIC ON THE FRONT OF OUR MINDS AND OMNICRON A BIG PART OF THIS, WE HAVE DIFFERENT LAYERS OF DATA AND THE RECORDS ARE NOT ACCESSIBLE. WE HAVE CLUNKY PORTALS AND THAT SORT OF THING BUT NONETHELESS, SOME DAY WE'LL HAVE AN IDEAL ELECTRONIC HEALTH RECORD OWNED BY THE PATIENT RATHER THAN THE HEALTH SYSTEM. THE IMAGES AND LABS AND GENOMICS THAT IS THE VIRUS AND THE HOST, THE OUTCOMES AND THE IMMUNOME AND WASTE WATER SURVEILLANCE ARE LAYERS WE CAN EACH HAVE DATA ON OUR SMARTPHONE WHICH WOULD BE REMARKABLE TO KNOW OF OUR INDIVIDUALIZED RISK AS WE GO ABOUT OUR BUSINESS AS WE GET TO AN ENDEMIC STATE OF THIS VIRUS OR FOR FUTURE PANDEMICS. NOW, I WANT TO GIVE ANOTHER EXAMPLE OF THIS MULTI-MODAL DATA. THAT'S WHAT WE'RE TALKING ABOUT, MULTI-DIMENSIONAL DATA AND ONE I'M EXCITE ABOUT WHICH HAS NOT BEEN ASSEMBLED BUT HAS EXTRAORDINARY POTENTIAL IS DIGITAL TWINS. WHAT I MEAN BY THAT IS IF YOU HAVE A DATA INFRASTRUCTURE SAY IT'S FOR CANCER WITH MILLIONS OF PEOPLE WHO HAVE HAD CANCER. IN THAT INFRASTRUCTURE YOU HAD ALL THE APPROPRIATE ELECTRONIC HEALTH RECORDS AND THE SCANS AND THE TUMOR SEQUENCING OF THE HOST ALL THE RELEVANT DATA AND THE TREATMENT AND OUTCOMES. NOW A NEW PERSON COMES ALONG WITH CANCER. INSTEAD OF THE CLINICALS WE HAVE TODAY OR COMPLIMENTING THE CLINICAL TRIAL WE HAVE IS BEING ABLE TO COME UP WITH A WHOLE NEW PATH OF PRECISION. THAT IS FINDING TWINS THAT MATCH UP AS CLOSELY AS POSSIBLE TO THE INDEX PATIENT AT HAND TO BE ABLE TO PREDICT THE TREATMENT AND OUTCOMES. WHY IS THIS SO IMPORTANT? FIRSTLY, THE CLINICAL TRIALS WE HAVE TODAY ARE REALLY POSITIVE CLINICAL TRIAL. SAY PAXLOVID FOR COVID THEIAN -- ANTI-COVID PILL GOING TO REVIEW HELPED 7 OF 100. THAT WAS 89 REDUCTION IN EFFICACY. WHAT ABOUT THE OTHER 93 OF 100 WHO DIDN'T DERIVE BENEFIT? I USE THAT EXAMPLE BECAUSE MOST CLINICAL TRIALS HAVE NOT 10 PER 100 AND WE'VE SEEN PEOPLE WITH SPECIFIC MUTATIONS IN THEIR CANCER CAN BENEFIT FROM TREATMENTS FROM OTHER PRIMARIES. THAT'S THE TIP OF WHERE IT CAN DO AND THINK OF IT NOT JUST FOR CANCER BUT PLANETARY RESOURCE ACROSS CONDITIONS. A WHOLE WAY TO TRY TO COME UP WITH THE BEST TREATMENT AND OUTCOMES THAT COMPLIMENTS THE EVIDENCE WE TODAY. I HAVE CASES OF MULTI-DIMENSIONAL EI. I MENTIONED SURVEILLANCE. A BIG AREA OF RESEARCH ARE THE DIGITAL CLINICAL TRIALS BECAUSE HERE WE'RE GETTING ACCESS WITH SENSORS AND CAN COLLECT BLOOD SAMPLES THROUGH MICRONEEDLE PATCHES AND CAN GET ENVIRONMENTAL DATA THROUGH SENSORS AND GET PEOPLE TO SAMPLE THEIR GUT MICROBIOME. WE CAN DO RAPID GENOME SEQUENCING WHERE THE DIAGNOSIS IS NOT IMMEDIATELY APPARENT. REMOTE MONITORING. THINK ABOUT THIS. WHAT ABOUT ALL THE PATIENTS THAT COULD STAY HOME IN THE COMFORT OF THEIR OWN HOME AT THE REDUCED EXPENSE WITH THEIR FAMILIES AND HAVE SENSORS AND ALGORITHMS TO PREDICT WHETHER THERE WAS IMMINENT ISSUE OF COMPROMISE WHERE THEY NEED TO COME TO THE HOSPITAL OR A TEAM DISPERSED TO THE PATIENT'S HOME. THAT WOULD REVAMP HOW WE USE HOSPITALS TODAY. I MENTION THE DIGITAL TWIN. AND THE LAST THING I WANT TO TOUCH ON IS THIS, FOR ALL OF TIME WE TALKED ABOUT PREVENTION BUT WE'VE NEVER REALLY ACTUALIZED IT IT'S SECONDARY PREVENTION. WHAT IF WE CAN HAVE PRIMARY PREVENTION BY HAVING THE DATA AND THE CORPUS OF THE MEDICAL LITERATURE PROFS PROCESSED IN REAL TIME AND GIVEN TO THE PERSON, IF THEY'RE WILLING AND TO HELP GUIDE THEM NEVER MANIFEST A CONDITION THEY HAVE HIGH RISK FOR DEVELOPING WHETHER THROUGH GENOMICS OR DIGITAL TWINS OR ANY OTHER MECHANISM. THIS IS EXCITING BECAUSE SOME DAY THIS WILL BE POSSIBLE. IT WILL TAKE SOME TIME. WE DON'T HAVE GOOD WAYS TO DEAL WITH IN STRUCTURED TEKZ BUT THAT'S IMMINENT IN THE YEARS AHEAD. THIS IS A PATH TO REAL PREVENTION TO FULFILL THE FANTASY THAT'S BEEN AROUND FOREVER. MULTI-MODAL A.I. IS THE TRANSFORMATIVE FUTURE AND IT'S ASSEMBLING THE PARTS. WE DON'T KNOW HOW TO DO THAT YET. NO ONE HAS DONE IT BECAUSE ESPECIALLY TAXING THIS ANALYTICS APPROACH IS THE CONTINUING DATA FROM SENSORS AND HOW IT CAN BE OPTIMALLY INTEGRATED. MANY GROUPS ARE WORKING ON MULTI-MODAL A.I. IN MEDICINE. IT'S AN EXCITING PATH AND PULLS TOGETHER WHAT WE DISCOVERED IN THIS PANEL AS WELL AS MANY OTHER THINGS TOUCHED ON THROUGHOUT THE NCATS 10th ANNIVERSARY. THANKS SO MUCH FOR HAVING ME. >> I'M EXCITED TO RE-INTRODUCE JONI RUTTER AND I'M INTRODUCE YOU PROPERLY AND JONI YOU HAVE A Ph.D. IN PHARMACOLOGY AND TOXICOLOGY. IT SEEMS AS YOU IF SPENT YOUR ENTIRE CAREER AT THE NATIONAL INSTITUTES OF HEALTH WHICH IS AMAZING. YOU DID AN NCI FELLOWSHIP EARLY ON AND THE LEAD OF THE DIVISION OF NEUROSCIENCE AND BEHAVIOR AT NIDA AND STARTED UP THE NEW PROGRAM CALLED "ALL OF US" AND WERE THE DIRECTOR OF SCIENTIFIC PROGRAMS. THEN I'M NOT SURE WHAT THE RECRUITMENT PROCESS LOOKED LIKE BUT ENTICED TO COME TO THE INSTITUTE WE'RE CELEBRATING 10 YEARS OF NCATS. YOU SERVED AT THE DEPUTY DIRECTOR AND NOW THE ACTING DIRECTOR. SO JONI, WHAT WAS IT LIKE THREE YEARS AGO WHEN YOU CAME FROM THE "ALL OF US" PROGRAM? WHEN WERE YOUR IMPRESSIONS NOW COMPARED TO WHY NCATS IS TODAY IN THE DEVELOPMENT JOURNEY, IF YOU WILL? >> YOU KNOW THE COMPARISON OF WHERE NCATS IS THREE YEARS AGO IS QUITE AMAZING. IT WAS AMAZING BEFORE BUT FOR THE BULK OF THE TIME I SPENT MORE THAN HALF MY TIME OUTSIDE OF AN OFFICE IN A BUILDING AND MORE OF THAT TIME AT HOME DUE TO THE COVID RESEARCH. THAT'S A CHANGE IN AND OF ITSELF AND THE SECOND THING IS THAT THE COVID PANDEMIC WAS SOMETHING THAT WE'VE ALL HAD TO PIVOT IN THIS NEW WAY BUT WHAT STRUCK ME ABOUT NCATS IS THE WAY NCATS THINKS AND HAS BEEN POSITIONED OVER THE COURSE OF THE LAST 10 YEARS IT'S ABOUT STUDYING DISEASE AND HOW TO APPLY TOOLS AND RESOURCE TO BRING TO BEAR ON THOSE DISEASES BUT WHEN THE PANDEMIC HIT ALL THOSE TOOLS AND RESOURCES THAT WE HAD AT OUR DISPOSAL WE WERE ABLE TO PIVOT AND BRING THEM TO THE PANDEMIC AND FROM THE PRECLINIC SPACE TO COMMUNITY ENGAGE THE WE'VE BEEN ABLE TO BRING ALL THAT POWER TO BEAR ON COVID-19 AND I THINK WHAT WAS INTERESTING ABOUT COMING TO NCATS FROM ALL OF US IS THIS IDEA THAT WELL AT ALL OF US IT'S A WAY OF THINKING ABOUT A PERSON AND OF COURSE TRANSLATING THAT TO A POPULATION. UNDERSTANDING WHAT A PERSON BRINGS TO THE TABLE IN OUTCOME AND BEHAVIOR AND ENVIRONMENT AND THE EXPERIENCE AND ALL OF US IS UNDERSTANDING THE WHOLE ENVIRONMENT AND THAT EXPERIENCE THEY HAVE. "ALL OF US" IS ABOUT UNDERSTANDING WHAT THE EXPERIENCE LOOKS LIKE TO COLLECT MORE INFORMATION TO DO BETTER PRECISION MEDICINE ON PEOPLE WHO HAVE SIMILAR EXPERIENCES LIKE THAT. THAT'S THE POWER OF ALL OF US. UNDERI ININ ININ INING -- UNDERSTANDING HO W TO GET THE ROUTINE INFORMATION OUT AND TO DO BETTER PRECISION MEDICINE REQUIRES US TO DO BETTER PRECISION MEDICINE RESEARCH. AND I THINK THE LEARNING FROM ALL OF US WILL HELP US TRANSLATE TO WHAT WE DO AT NCATS. >> I LOVE HEARING YOU TALK ABOUT THE SYNERGY BETWEEN WHETHER WE'RE TALKING ABOUT "ALL OF US" AND THINGS YOU MINUTED AT NIDA AND THE OTHER SPACES OF NIH, ACTIVE THE COVID RESPONSE AND I THINK THE SYSTEMS LEVELS THINKING IS WHAT IS NEEDED IN TERMS OF WHAT WE'RE GOING TO SEE IN THE FUTURE AND FRANCIS AND OTHER PEOPLE HAVE SAID AND THERE WAS A BOOK ABOUT IT WE DON'T GET DISEASES ONE INSTITUTE AT A TIME. THE ABILITY TO CREATE THE TEAM SPORT ATMOSPHERE IT'S PALPABLE IN WITNESSING HOW YOUR TEAM AND STAFF AND LEADERS REPRESENT AND INTEGRATE INSIDE AND HATS OFF A PERSONAL THANK YOU TO YOU FOR YOUR AMAZING LEADERSHIP. I WANT TO ASK YOU ONE MORE QUICK QUESTION BEFORE I HAND IT TO YOU FOR YOUR FINAL TALK. I KNOW WHEN YOU HAD SOMETHING CALLED FREE TIME BEFORE THE PANDEMIC HIT, I THINK YOU'RE FAVORITE HOBBY WAS BIRD WATCHING. ANY PARALLELS BETWEEN WHAT YOU DO WHEN WHAT YOU DO AS A BIRD WATCHER AND NCATS. ANYTHING THERE THAT STRIKES YOU AS SIMILAR. >> THAT'S A GREAT QUESTION. WHAT I LIKE ABOUT BIRD WATCHING I MAKE IT A CHALLENGE. IT'S NOT SUPPOSED TO BE A CHALLENGE BUT IT'S A RELAXING HOBBY BUT WHEN I HEAR I BIRD I LIKE TO BE THE FIRST ONE TO FIND THE BIRD. THIS IS A COMPETITION WITH MY HUSBAND SO IT IS KIND OF LOCAL. THAT'S WHAT I FIND FASCINATING. HOW DOES MY BRAIN INTERPRET THE DATA I'M HEARING FROM THE BIRD AND ALLOW ME TO THEN GO SEE AND FIND THE BIRD AND SEE THE BIRD AND PUT EYES ON IT AND IT'S SIMILAR TO WHAT WE DO AT NCATS. I LEARNED QUICKLY EARLY ON I DON'T HAVE ALL THE ANSWERS WHEN YOU'RE TRAINED UP IN A PARTICULAR DISCIPLINE YOU WON'T HAVE ALL THE ANSWER. ONE OF THE BIGGEST SKILLS IS BRINGING PEOPLE TOGETHER TO ADVANCE THE PROGRAMS. THIS WAS ACUTE FOR ME EARLY ON AND AS A GENETICIST BECAUSE WHEN I WOULD FIND A GENE THAT MIGHT CAUSE A DISEASE OR WORK WITH A TEAM THAT CAUSES A DISEASE MY SKILLS WERE CALLED TO ANOTHER DISEASE AND WORKING WITH ANOTHER TEAM AND WHAT I WANTED TO DO WAS MOVE FROM THE ONE DISEASE TO SOMETHING WE COULD APPLY MUCH MORE BROADLY AND THINK ABOUT HOW TO INCORPORATE DATA TO FIND OUR TARGET. THAT WILL DRIVE THE IDEAS HOME TO FIND MORE TREATMENTS. THAT'S WHAT I LOVE ABOUT NCATS WITH THAT FUZZY RELATIONSHIP TO BIRDING. >> I LOVE THE GATHERING ALL THE INSIGHT YOU SHARED AND THE COMPETITIVE NATURE. IF THERE'S A TRIVIA NIGHT I'M ON YOUR TEAM. JONI, I THINK YOU'RE DOING A GRAND SUMMARY TALK FROM THIS AMAZING DAY OF PRESENTATIONS. HATS OFF TO YOU AND TO THE TEAM AT NCATS AND THE NETWORK YOU ALL ARE SOMETHING AND HAPPY BIRTHDAY OFF TO THE SCIENCE OF THE FUTURE AND THANK YOU FOR HAVING ME HERE TO HELP YOU CELEBRATE. >> THAT WAS TERRIFIC. I APPRECIATE YOU JOINING US AND I THINK I CAN SPEAK AT NCATS SAYING HOW MUCH WE HAVE APPRECIATED YOUR PARTNERSHIP AND GUIDING US OVER THE LAST DECADE. YOU'VE BEEN THERE FOR US AND I'M SO COMBLAD YOU WERE -- GLAD YOU WERE ABLE TO MAKE IT TODAY. >> THANKS, JONI. >> HELLO AGAIN, EVERYONE. WOW, WHAT A RICH THOUGHT-PROVOKING AND FUN DAY. THE AFTERNOON FLEW BY FOR ME AND BEFORE I GET STARTED I WANT TO THANK A VARIETY OF PEOPLE BECAUSE I WANT TO MAKE SURE I HIT THAT UP FRONT. IT'S SO IMPORTANT TO BRING EVERYONE TODAY AND THERE'S PEOPLE WANT TO THANK FOR MAKE THE 10th ANNIVERSARY VERY WELL BRACE HAPPEN. I WANT TO THANK FRANCIS COLLINS AND MARGARET FOR THEIR INSIGHT. IT WAS HELPFUL TO HEAR AND AND A SHOUT OUT TO OUR NCATS STAFF. YOU GOT TO HEAR FROM THE STAFF BUT FOR EVERY ONE OF THEM THERE'S MANY MORE WHO TIRELESS CONTRIBUTE AND THAT'S OUR SECRET SAWS THE PEOPLE. THANK YOU TO THE SPEAKERS AND THEY PACKED A BUNCH AND SHOWED TRANSLATIONAL SCIENCE APPROACHES WORK AND THEY'RE ROBUST AND THE TALKS COVERED A VAST RANGE OF RESEARCH. I WANT TO THINK OF THE ACCOMPLISHMENT AND TALKED ABOUT THE DATA AND WE TALKED ABOUT THE TRANSLATOR AND HOW IT'S SAVING LIVES AND SAID DATA IS THE PIVOTAL FORCE IN ALL TRANSLATIONAL PROJECTS AND THAT'S SO TRUE. HE THEN TOLD US WHY THROUGH A POWERFUL STORY ABOUT HIS SON, BERTRAND AND WAS THE FIRST PERSON DIAGNOSED WITH THE NGL1 DEFICIENCY AND HE SEARCHED SYMPTOMS WITH PATHOGEN DATA AND PUTTING THE DATABASES TOGETHER AND UNDERSTANDING THE POWER OF THE DATA HE WAS ABLE TO IDENTIFY A PATH FOR A TREATMENT APPROACH THAT GAVE PBERTRAND 509 EXTRA DAYS AND MATT THANK YOU FOR SHARING THAT STORY ABOUT BERTRAND. THEN WE HEARD SIMILAR STORIES FROM THE I'D QUA OF THE PROMISING PRE-CLINICAL SECTION AND DURING THE PROMISE IING PRE-CLINICAL CANDIDATES AND WE HEARD ABOUT ANOTHER KID WHO IS ONE OF 10 IN THE WORLD WITH THE DISEASE AND WE FOUND OUT IF HE COULD HAVE HELP WHAT TO DO ABOUT THE DISEASE AND WHAT HE FOUND WAS NOT JUST HELP BUT HOPE AND HOPE IS VERY IMPORTANT. AND TIM YU TALKED ABOUT THE RARE DISEASE AND THE SILENT EPIDEMIC AND THINKING ABOUT ALTERNATIVE APPROACHES TO ADDRESS THE IDEA FEW PATIENCE WHO HAVE ULTRARARE DISEASES DON'T HAVE TO BE IGNORED JUST BECAUSE THERE'S A FEW PEOPLE WITH THOSE DISEASES. DR. YU COLLABORATED WITH A CHILD NAMED MILEA AND THEY ALL COLLABORATED TO THINK ABOUT GENERATING GENE TARGETED THERAPIES AND PLATFORM TECHNOLOGIES RATHER THAN CREATING AN APPROACH FOR ONE DISEASE AT A TIME. THEY WERE ABLE TO TREAT MILEA WITH A NEW THERAPY CREATED JUST FOR HER IN ONE YEAR BUT THE POWER OF THAT WAS THE APPROACH THEY USED CAN BE APPLIED TO OTHER DISEASE AND HE SAID WE NEED TO TAKE CREATIVE AND PRODUCTIVE RISK IN DRUG DEVELOPMENT TO MAKE THOSE ADVANCEMENTS. SOMETIMES IT'S NOT ABOUT THE MOLECULE BUT THE PLATFORM TO PROPEL MORE TREATMENTS TO FOLLOW. ON THE NEXT SESSION CHRISTINA HARTMAN TALKED ABOUT DISEASE CAN BE PLUG AND PLAY AND THE SAME IDEA OF TREATING MORE THAN ONE DISEASE AT A TIME WHERE ED SAID WE NEED RARE DISEASE PENICILLIN TO CURE MULTIPLE DISEASES AND WE HEARD ABOUT THE BESPOKE GENE CONSORTIUM THROUGH THE NIH ACCELERATED MEDICINES PROGRAM AND TALKED ABOUT THE IDEA OF AAV VECTORS AS MULTI-PURPOSE DEVICES MAKING EQUITABILITY OF ACCESS TO THOUSANDS OF ALL ULTRA RARE APPRECIATES AND WE HEARD THE FUTURE OF CLINICAL RESEARCH MUST REDUCE COSTS AND LANGUAGE BARRIERS INCLUDING CULTURAL ADAPTATION ENSURING INCLUSIVITY. FOR SERVICES THAT ARE SUSTAINABLE AND COMMUNITY ENGAGE THE IS CRITICAL AND THE COMMUNITY HAS A PROBLEM. WE HAVE TO WORK WITH THE COMMUNITY TO PROVIDE SOLUTIONS. THOSE ARE WISE WORDS AND ON THE DATA SIDE WE HEARD ABOUT MULTI-MODAL DATA TO COME UP WITH A WHOLE NEW PATH OF PRECISION MEDICINE LIKE USING DIGITAL TWINS AND DATA FROM EXISTING RESOURCES THAT HAVE CHARACTERISTICS AND HOW CAN THAT POWER DATA HELP FIND NEW PATHWAYS FOR TREATMENT. AND I WANT TO END WITH THINGS DR. COLLINS MENTIONED AT THE TOP OF THE MEETING HE SAID WE CAN'T JUST HOPE ADVANCES WILL HAPPEN. WE NEED TO MAKE IT HAPPEN. AND IF YOU WANT TO GET SOMETHING DONE YOU WANT TO SURROUND YOURSELF WITH SMART PEOPLE. THAT SPILLS THE BEANS AS TO WHY THIS EVENT IS SO IMPORTANT TO ME AND TO NCATS AND OUR STAFF. WE'RE BRINGING PEOPLE TOGETHER TO HELP US FORGE AHEAD TOGETHER. SO THIS IS A LOT OF TO BUILD UPON AND WHAT CAME THROUGH IS WHAT WE VALUE STRONG COLLABORATIONS WORKING WITH A COMMUNITY OF SCIENTISTS AND ADVOCACY ORGANIZATIONS AND PATIENT TO GATHER INPUT AND LETTING SCIENCE LEAD THE WAY AND IT'S HARD NOT TO BE INSPIRED BY THE FABULOUS TALKED WE HEARD ABOUT TODAY. IT'S ALSO WHAT WE NEED TO THINK ABOUT IN TERMS OF THE NEXT DECADE AND THAT'S WHAT I WANT TO CLOSE ON. THE LAST PIECE IS ABOUT WHAT'S NEXT. TRANSLATIONALITY -- TRANSLATIONAL SCIENCE CAN HELP BRING MORE TREATMENTS TO MORE PEOPLE MORE QUICKLY. AND THIS ISN'T JUST OUR MANTRA, THIS IS OUR VISION. IT'S OUR HILL TO CLIMB AND THINK WHAT WE'LL BE ABLE TO SEE FROM THE NEW HEIGHT BECAUSE OF WHAT WE WILL HAVE LEARNED AND OVERCOME ALONG THE WAY WHEN WE REACH THAT HILL. AND TO TAKE THE HILL WE'LL NEED GOALS AND I'LL LAY OUT THREE GOALS. MORE TREATMENT, ALL PEOPLE, MORE QUICKLY. GOAL NUMBER ONE, MORE TREATMENT. RIGHT NOW WE KNOW 5% OF DISEASE HAVE A TREATMENT SO LET'S GET THAT UP SO A QUARTER OF KNOWN DISEASES HAVE A TREATMENT IN THE PIPELINE. WE ALSO KNOW THAT 90% OF DRUGS IN PRECLINICAL DRUGS FAIL BECAUSE OF LACK OF EFFICACY AND IN VITRO AND ANIMAL MODELS ARE NOT RELIABLE. THEY'RE NOT PREDICTIVE WHETHER A DRUG WILL BE PREDICTIVE IN HUMANS AND WHAT WE HEARD IS IN VITRO ASSAYS THAT RECAPITULATE HUMAN PHYSIOLOGY SHOW PROMISE FOR MORE PREDICTIVE OF EFFICACY IN HUMANS. WE NEED TO ENABLE THE PLATFORM TECHNOLOGIES LIKE TISSUE CHIPS AND 3-D MODELS TO HAVE A WIDE ARRAY OF ORGANS THAT MODEL THE DIVERSITY OF INDIVIDUALS AND GENETIC MAKEUP AND ENVIRONMENTAL EXPOSURES AND THE TIP MAKING THEM MAINSTREAM AND NEED TO PUSH FOR ACTIVE DISSEMINATION AND USE SO THE TOOLS DON'T JUST SIT ON THE SHELVES. THEY NEED TO BE TRIED AND TREESTED TO GET TO NEW TREATMENTS AND THE SOMATIC CELL GENE EDITING PROGRAM AND OTHERS ARE POISED TO TRANSFORM THE PLATFORM FOR BREAKING DOWN THE BARRIERS OF MANUFACTURING AND REGULATORY HURDLES THAT HAVE SLOWED US DOWN TO GET US TO MOVE FORWARD. WE KNOW 80% OF 7,000 RARE DISEASE SINGLE GENE DISORDERS AND IF THEY WORK FOR LESS THAN HALF OF RARE DISEASES THAN HUNDREDS OF RARE DISEASES AND THOUSAND OF RARE DISEASE PATIENT WE HAVE HOPE FOR. WHILE THE PROGRAMS FOCUS ON A HANDFUL OF RARE DISEASES OUR RESOURCES WILL HELP RESEARCHERS WORLDWIDE IN THERAPIES AND TECHNOLOGIES. WE CAN DEMOCRATIZE THE TECHNOLOGY. THESE PROGRAMS ARE NOW NCATS WORKS WITH INDUSTRY, ACADEMIA, PATIENT AND PATIENT ADVOCACIES GROUPS AND ALL OTHERS AND WORKING TO DEATH TO IDENTIFY CAUSING THE SLOW PACE AND CREATING PLATFORM TO HELP FIX THE CRIMPS TO BRING CHANGE TO WHAT'S NEEDED AND THIS MODEL WORTH REPEATING TO GET US TO MORE TREATMENT. SO GOAL NUMBER 2, ALL PEOPLE. WE NEED TO DRAMATICALLY INCREASE INCLUSIVITY IN ALL AREAS INCLUDING WORKFORCE, TRAINING, RESEARCH, CLINICAL OUTCOMES FOR THOSE IN ETHNIC PROPOSITIONS AND RURAL AREAS ARE ONE OF THE MOST PAINFUL AREAS OF THE PANDEMIC AND BLACK AND AFRICAN AMERICAN AND LATINOS AND OTHERS ARE DYING AT HIGHER RATES THAN WHITE AND ASIAN AND IN RECENT DATA FROM THE ENCLAVE WE HEARD ABOUT TODAY SHOWED MORTALITY RATES IN RURAL COMMUNITIES ARE 40% HIGHER THAN IN URBAN COMMUNITY. WE NEED TO CONTINUE TO ADDRESS THE DISPARITIES. AND THE TSA PROGRAM HAVE MADE APPROACHES HAVE ENABLED THEM TO PIVOT RAPIDLY TO ADDRESS COVID-19 DISPARITIES. THERE'S MORE WORK TO DO. STILL THE NUMBER ONE PROBLEM IN COMPLETING A CLINICAL TRIAL IS RECRUITMENT. THE CTSAs ARE CONNECTING WITH UNDER SERVED AND VULNERABLE INNOVATIONS AND TRYING TO MEET PEOPLE WHERE THEY ARE USING MOBILE LAND AND GROCERY STORES GOING TO LIBRARIES AND PLACES OF WORSHIP AND HELPING IMPROVE HEALTH OUTCOMES FOR THOSE DISPROPORTIONATELY AFFECTED FOR COVID-19 AND WILL BE NEEDED TO ENSURE NO ONE IS LEFT BEHIND IN OUR PLANNING AND DESIGNING AND IMPLEMENTING TO ENSURE THE RESEARCH IMPACT IMPROVES LIVES FOR US ALL. AND COVID-19 IS NOT THE ONLY EXAMPLE OF INEQUITY. THERE'S MANY AND WE HAVE TO DO BETTER. THINK ABOUT SICKLE CELL DISEASE. IT WAS THE FIRST GENETIC DISEASE IDENTIFIED WAY BACK IN 1910. BUT ONLY NOW ARE WE BEGINNING TO SEE THE CURES FOR THE THOUSANDS OF BLACK AND AFRICAN AMERICANS WHO SUFFER FROM THAT DISEASE AND SPEAKING OF SICKLE CELL DISEASE IT'S ALSO A RARE DISEASE AND THOSE WITH ASSOCIATED DISPARITIES ALONG WITH BEING UNDER STUDIED BECAUSE THEY'RE RARE. WHEN IT COMES RARE DISEASES THERE'S MORE THAN 7,000 OF THEM AND DEFINED AS FEWER THAN 200,000 WITH RARE DISEASE. MOST DON'T COME CLOSE TO THE PREVALENCE OF 200,000. MANY ARE FEWER THAN 100 INDIVIDUALS. MOST ARE ULTRA RARE. BECAUSE THERE'S SO FEW THERE'S LITTLE COMMERCIAL INTEREST TO FIND TREATMENT FOR THEM. EVEN SO, THE PUBLIC HEALTH BURDEN OF RARE DISEASES IS $400 BILLION IN ANNUAL MEDICAL CARE COSTS PER YEAR. THAT'S HIGHER THAN ALZHEIMER'S DISEASE, HEART DISEASE AND CANCER AND RESEARCH WILL LEAD TO MORE COMMON DISEASES AND RARE DISEASES ARE NOT RARE. FUNDING FOR RARE DISEASE RESEARCH SHOULD NOT BE RARE EITHER. SO INCREASING DIVERSITY AND APPROACH TO HELP TO GET TO ALL PEOPLE. GOAL 3, I MENTIONED EARLIER 90% OF DRUGS IN THE PIPELINE FAIL. I NEED TO ADD IT TAKES 10 TO 15 YEARS AND ABOUT 2. BILLION FOR A DRUG TO MAKE IT TO MARKET. WE NEED TO REDUCE THAT TIME BY HALF. FAIL FAST, FAIL EARLIER FOR EXAMPLE USING THE BETTER PRE INDICATIVE MODELS AND PATIENT-CENTERED DESIGN AND NEED MORE STRATEGIES TO STUDY MANY DISEASES AT A TIME TO FIND WHAT'S COMMON AND IDENTIFY THE COMMON TARGET AND UNDERLYING MECHANISM AND TRAIN THE NEXT GENERATION OF SCIENTIST TO EMBRACE TEAM SCIENCE AND THINK IN TERMS OF NOT JUST EVOLUTION ABOUT THE REVOLUTIONARY TRANSFORM AND IT'S NOT ALWAYS ABOUT THE SCIENCE. WE NEED TO CHANGE WHAT'S NOT WORKING. IF IT'S SLOWING YOU DOWN USE THAT TIME TO STREAMLINE SO YOU CAN SPEED UP THE NEXT TIME. WE HEARD A LOT ABOUT TEAM SCIENCE TODAY AND COLLABORATIONS AND PARTNERSHIPS ARE CRITICAL FOR OUR SUCCESS AND WITH MORE THAN 150 ACTIVE RESEARCH COLLABORATIONS WIN COLLEAGUES AT NIH AND BEYOND, OUR OFFICE OF STRATEGIC ALLIANCES HAVE DEVELOPED AGREEMENT THAT OTHERS CAN USE TO QUICKLY LAUNCH COLLABORATIONS IN WEEKS NOT MONTHS. USING SMART IRBs AS MENTIONED EARLIER TO SET UP CLINICAL TRIALS IN A MATTER OF MINUTES NOT MONTHS. SO WHAT ARE PROCESSES LIKE THAT YOU CAN HELP CHANGE? AND THEN THERE'S DATA. THERE'S TRANSFORMATIONAL POWERS FOR TESTING AND USING HIGHER POWERED A.I. TO SEE PATTERNS IN DATA WE COULDN'T OTHERWISE AND IT'S ABOUT HARNESSING THE POWER OF THE DATA AND MATT HALL TALKED ABOUT DRUG REPURPOSING TO FIND NEW THERAPEUTICSES AND DRASTICALLY CUTTING DOWN THE TIME IT WOULD TAKE SOMETHING TO GET SOMETHING CLEARED FROM 10 TO 15 YEARS TO ONE YEAR AND SPEEDING UP FIRST CANDIDATE AND YOU HEARD ABOUT THE ELECTRONIC HEALTH RECORDS DATABASE THE COLLABORATIVE OR N3C WHERE HUNDREDS OF COLLABORATORS MADE THIS NATIONAL RESOURCE POSSIBLE AND TOGETHER DELIVERED ON ITS PROMISE OF TRANSFORMING CLINICAL DATA INTO ACTIONABLE KNOWLEDGE AND RESEARCHERS ARE HARNESSING THE POWER FOR COVID-19 AND WILL INFORM DIRECTION WHILE FOLLOWING THE HIGHEST FORMS OF SECURITY AND PRIVACY. AND WE'RE NOW TALKING ABOUT QUANTUM COMPUTING. THESE ARE STILL FIVE TO 10 YEARS DOWN THE ROAD BUT NOW IS THE TIME TO MAKE SURE WE'RE READY TO USE THE TOOLS WHEN THEY MAKE THEIR QUANTUM LEAP. THESE ARE THE KINDS OF APPROACHES TO GET US FROM A TO B MORE QUICKLY. THESE ARE THE GOALS AND THEY'RE INSPIRATIONAL AND WILL GUIDE US TOO THE NEXT DECADE AND KEEP US FOCUSSED ON OUR VISION OF MORE TREATMENTS TO MORE PEOPLE, MORE QUICKLY. WE HAVE IMNUMERABLE STAKEHOLDERS WHO RELY OUNCE AND WHAT WE DO AND CAN PIVOT ON A DIME TO ADDRESS A VARIETY OF DREAMS AND EVEN PUBLIC HEALTH EMERGENCIES LIKE ZIKA AND EBOLA AND COVID. COVID IS AN FORMIDAB ABLABLE OPPONENT AND SO ARE WE AND YOU'RE THE CATALYST. WE ALL WORKED SO HARD TO ADDRESS COVID-19 AND WINNING THE FIGHT FOR ALL DISEASES WILL REQUIRE THE SAME INTENSE AND SAME COMMITMENT AND ENERGY. WE HAVE BURN THE CANDLE AT BOTH ENDS TO SHED LIGHT ON COVID TO UNDERSTAND HOW TO DEFEAT THE VIRUS AND HOW IT MOVES THROUGH THE POPULATION. WHAT INTERVENTIONS MIGHT HELP BUT WE'RE READY FOR THE NEXT CHALLENGE. SO THANK YOU ALL FOR MAKING NCATS' EXTRAORDINARY 10-YEAR JOURNEY POSSIBLE AND THANK YOU FOR SHARING YOUR VISION, DEDICATION AND PERSPECTIVES AND LONG DAYS AND SO MUCH OF YOURSELVES. TRANSLATIONAL SCIENCE IS TEAM SCIENCE AND COLLECTIVELY YOU'VE HELPED PUSH US ALL RATHER THAN INDIVIDUAL AND BRINGING MORE TREATMENT TO MORE PEOPLE MORE QUICKLY AND THAT'S OUR HILL AND WE NEED YOUR HELP TO CLIMB IT AND HOPE TO MEET YOU AT THE TOP FOR THE DECADES TO COME AND THANK YOU AND HAPPY BIRTHDAY NCATS.