>>I WILL GO AHEAD AND GAVEL US IN FOR THE 30th MEETING OF THE NATIONAL CENTER FOR ADVANCING TRANSITIONAL SCIENCES ADVISORY COUNCIL. WELCOME. TAKE IT AWAY. >> . >> I WILL START WITH THE ROLLCALL. [ROLL CALL] >> WE HAVE A FULL COMPLEMENT OF MEMBERS TODAY. IN THE SLIDE YOU CAN SEE THE FIRST DAY OF THE TODAY MEETING CALLED TO ORDER I WILL DO SOME ANNOUNCEMENTS AND WE'LL HAVE THE DIRECTORS REPORT AND A PRESENTATION AND THEN A PROGRAM UPDATE FOR CLINICAL INNOVATION. AND WE WILL RECONVENE 1:00 O'CLOCK P.M. TOMORROW DAY TO OF THE OPEN SESSION. THE INVITED SPEAKER TODAY CHIEF OFFICER OF SCIENTIFIC WORKFORCE DIVERSITY AT NIH WILL BE DELIVERING A PRESENTATION TO ENSURE THAT ALL ARE OUT AT THE TABLE. AFTER WE HAVE THE INVITED PRESENTATION WE WILL HAVE THE PROGRAM UPDATE DELIVERED BY THE DEPUTY DIRECTOR OF PROGRAMS FROM THE DIVISION OF PRECLINICAL INNOVATION. TOMORROW THE PROGRAM UPDATE WILL BE THE DIRECTOR OF THE OFFICE OF SPECIAL INITIATIVES AND IN SEVERAL PRESENTATIONS TOMORROW. ANNOUNCEMENTS. >> EVERYONE IN THE ZOO MEETING IS A PANELIST TODAY YOU'RE FREE TO SPEAK HOWEVER TO MAINTAIN A SMOOTH FLOW PLEASE USE THE RAISED HAND FEATURE ON —- FEATURE AND THEN WAIT UNTIL YOU ARE CALLED UPON. BUT THE SPEAKERS TO THE PRESENTATIONS WE ARE USING THE VIDEOCAST FEATURE TODAY AUDIENCE MEMBERS CAN SUBMIT COMMENTS THROUGH JUNE 4th BEING ON THE SITE OR THE ADDRESS THAT YOU SEE ON THE SLIDE. PLEASE MAKE SURE WHEN SPEAKING YOUR CAMERA IS ON FOR THOSE WHO VIEW BY VIDEO CAN SEE THAT YOU ARE SPOTLIGHTED IF YOU ARE NOT IN CAMERA THE PREVIOUS SPEAKER REMAINED SPOTLIGHTED. NOW LOOKING AT THE JANUARY 2022 MEETING PROVIDED TO THE NUMBERS CAN I HAVE A MOTION TO APPROVE THE OF THE JANUARY MEETING? >> I SECOND. ALL IN FAVOR? ALL THOSE OPPOSED? >> I HAVE STAYED I HAVE NOT READ THE MINUTES. >> THANK YOU. I DO BELIEVE ALL MEMBERS OF HAD AN OPPORTUNITY TO READ THE MINUTES AND A SUFFICIENT NUMBER DID SO. THANK YOU. NEXT SLIDE. THE FUTURE MEETING DATES FOR THE COUNCIL SEPTEMBER 22nd OF THIS YEAR A VIRTUAL ONE DAY MEETING AND THEN THE MEETINGS WILL BE JANUARY 26. THAT WILL BE VIRTUAL. MAY 25th AND SEPTEMBER 28 AND 2024 JANUARY 18 AND 19. THAT WILL BE VIRTUAL AND MAY 23d AND SEPTEMBER 26. WOULD LIKE TO TURN IT BACK TO DELIVER THE DIRECTORS REPORT. >> . >> . >> WHILE SHE IS DOING THAT WORD YOU MIND THOSE DATES AND BY SO QUICKLY INTO THE CHAT SOMEWHERE? I JUST WANT TO MAKE SURE IT'S IN MY CALENDAR. >> WILL MAKE SURE THE DATES ARE IN THE CHAT. >> YOU DO HAVE CONTROL OF THE MINUTES. WE CAN GET STARTED. WELCOME AND IT IS THE 30th MEETING THIS CAPS OFF OUR ANNIVERSARY. GOING FORWARD IF THIS WORKS I WOULD LOVE YOUR FEEDBACK I AM USING A HIGH TECH AND INTERACTIVE APPROACH FOR THE SLIDES TODAY IF YOU HAVE THE CELL PHONE ON HIS FIRST SLIDE YOU CAN USE IT TO TAKE A PICTURE OR HOLD YOUR CAMERA UP TO THE QR CODE. THAT WILL TAKE YOU TO THE WEBPAGE LANDING PAGE I SHOW YOU THE SLIDES BUT YOU CANNOT CLICK ON THEM SO I'M HOPING THAT THIS WILL GIVE A BETTER SENSE THIS IS THE HIGH TECH INTERACTIVE VERSION - - VERSION OF OUR MEETING. SO TODAY ALSO WE SAY GOODBYE TO ONE OF OUR OWN REVIEW BOARD MEMBERS MICHAEL HAS BEEN OUR MEMBER ON THE REVIEW BOARD SINCE 2019 THROUGH NOW AND HE HAS BEEN PART OF THE GROUP THAT SUPPORTED TWO MAJOR INITIATIVES INCLUDING GENE THERAPY INITIATIVE AND THE DRUG REPURPOSE SEEING INITIATIVE. MIKE IS HOW THE VARIETY OF LEADERSHIP ROLES IN INDUSTRY AND PHARMACY AND CURRENTLY THE SENIOR PARTNER AT FLAGSHIP PIONEERING AND CAMBRIDGE IT WILL CONTINUING IN THAT ROLE I WOULD LIKE TO THINK HIM FOR HIS SERVICE OVER THE YEARS AND WE DID FAREWELL AND THANK YOU FOR YOUR SERVICE. FOR THE REMAINDER OF THE TALKING WANT TO OUTLINE A VARIETY OF TOPICS AS WE MOVE THROUGH TO KEEP TRACK IN THIS WAY WITH NIH UPDATES WE WILL TALK ABOUT THE BUDGET THERE IS A LOT TO TALK ABOUT. I WILL ALSO GIVE YOU AN OVERVIEW OF THE PROBLEMATIC UPDATES I WILL BROWN THAT OUT FOR THE DIVERSITY EQUITY AND INCLUSION AND REQUEST FROM THE COUNCIL FROM THE LAST TIME ON THE TRY ANNUAL REPORT WHAT WE ARE DOING TO MITIGATE THOSE MEASURES OF THAT REPORT I WILL REALLY FOCUS MY EFFORTS ON THOSE ACTIVITIES CLEAR WILL MODERATE THE SESSION SO PLEASE FEEL FREE TO BUT THEM IN THE CHAT OR USE THE RAISED HAND FUNCTION SO IN TERMS OF ANNOUNCEMENTS AND ACTIVITIES RELATED TO NIH THERE ARE VARIETY OF LEADERSHIP SEARCHES. AS YOU KNOW FRANCIS COLLINS STEP DOWN DECEMBER 2021 AND LARRY TAILBACK HAS BEEN THE ACTING NIH DIRECTOR AND THAT IS STILL AN OPEN LEADERSHIP POSITION CURRENTLY UNDERWAY. WE HAVE NO NEWS TO UPDATE AT THIS POINT. THAT IS A PRESIDENTIAL APPOIN APPOINTEE. WE WILL AWAIT FOR THE WHITE HOUSE TO MAKE THOSE ANNOUNCEMENTS. OUR BUT AGE WAS TO BE UNDER THE RUBRIC OF HHS AS WELL AS NIH SO THAT IS IN EITHER ONGOING LEADERSHIP SEARCH HOPEFULLY THAT IS ALSO PRESIDENTIAL APPOINTEE THAT DOES NOT CURRENTLY REQUIRE SENATE CONFIRMATION AND THEN THERE ARE A VARIETY OF OPENINGS WITHIN THE NIH LEADERSHIP POSITIONS ACROSS THE INSTITUTES MEANT TO HAVE A VACANCY THE ACTING DIRECTOR SINCE HE HAS STEPPED DOWN AND THE NIH DIRECTOR HAS STEPPED DOWN. LIKE ME REMAINING IN THAT POSITION UNTIL THE DIRECTORS NAMED. I MENTIONED ARPA-H I WANT TO GIVE YOU A SENSE OF WHERE YOU ARE AND WHAT WE KNOW. AND WITH THOSE SESSIONS THAT WENT ON AND IN MARCH CONGRESS PASSED THE OMNIBUS THAT ALLOWS $1 BILLION FOR THREE YEARS FOR OUR PAGE TO START ACTIVITIES AND TO BE PRESIDENTIALLY APPOINTED AND THEN HIRING THOSE FLEXIBILITIES SO IT IS MEANT TO BE THAT ARE BUT AGE WILL BE COMPLEMENTARY AND NOT DUPLICATE OF NIH PROGRAMS SO I THINK THAT WILL BE VERY IMPORTANT TO SEE HOW THAT CONTINUES TO EVOLVE AND THEN PASSING THE OMNIBUS THERE IS LANGUAGE THAT DELEGATED THE RESPONSIBILITY OF WHERE ARPA-H WORD AND ABOUT THAT DISCUSSION OF HHS SECRETARY AND THEN TRANSFERRED TO NIH THAT IS THE HOME FOR THE PROGRAM GOING FORWARD AND IN THE PRESIDENTS BUDGET THERE IS $5 BILLION SUGGESTED FOR OUR PH HFI 23. MANY OF YOU ARE AWARE BUT YOU HAVE HEARD A NEW BILL THAT COULD MOVE IT BACK INTO HHS STAY TUNED OF COURSE THIS IS EVOLVING BUT THIS IS WHERE WE ARE NOW AND WE WILL CONTINUE TO PROCEED AS SUCH ANOTHER ANNOUNCEMENT IN TERMS OF THE OMNIBUS FROM NIH INSTITUTIONS MANDATORY WHEN IT COMES TO HOSTILE WORKING CONDITIONS AND PREVIOUSLY NIH LACKED CLEAR AUTHORITY TO REQUIRE FUNDED INSTITUTIONS TO REPORT NIH WHETHER PERSONAL CHANGES WERE RELATED TO HARASSMENT THE PROVISION THAT THEY SHOULD BUT NOT REQUIRED TO SO PASSAGE OF THIS BILL ENSURES THAT WHEN THE PERSONAL CHANGES IS DUE TO HARASSMENT AND THEREFORE STRENGTHENS THE ABILITY TO TAKE NECESSARY ACTION TO ENSURE SAFE WORK ENVIRONMENTS WHENEVER THOSE ACTIVITIES ARE CONDUCTED THIS GIVES US MORE TEETH IN THE SPACE AND HERE IS THE QR CODE. >> THERE ARE LONG-STANDING POLICIES ALL PROTECTING THE INFORMATION FROM THOSE PROVIDING AND IT HAS BEEN UPDATED AS A SPEAK AND WITH THE NEW POLICIES AND WHEN THEY ARE COMING INTO PLAY. AND THEN TO ENSURE ROBUST AND OF THAT DATA THAT'S IN AND THEN TO PUBLIC TRUST AND RESEARCH MATERIALS SO OBVIOUSLY THIS IS AN IMPORTANT PIECE OF WORK THAT THE NIH SUPPORTS AND I'M ALSO PROVIDING FOR A QR CODE WITH THE EFFORTS AROUND THIS SPACE AND THE DEADLINES THAT WILL BE POSTED ON THIS SITE. AS IT STANDS NOW YOU BE PROVIDING SUPPLEMENTAL INFORMATION AND PRIVACY PROTECTIONS AS WELL AS SAMPLE PLANS AND ADDITIONAL WEBINARS AS WELL. ONCE A POLICY TAKES EFFECT WE'LL BE DOING ON BOARD ASSESSMENTS TO PROVIDE INCENTIVES THAT IT HAPPENS IN THIS WAY. I ENCOURAGE YOU TO LOOK MORE DEEPLY AT INFORMATION PROVIDED ON THE WEBSITE. SO NOW WE WILL TURN TO THE EVENTS AND OTHER ANNOUNCEMENTS. I WILL START OFF BY TALKING ABOUT THE ORGANIZATIONAL CHANGES EVOLVE AND AWARE WE WANTED TO CHANGE OUR OFFICES TO ELEVATE TO HER THEY STAND WITHIN THE NIH IN TERMS OF OTHER DIVISIONS WITHIN OUR STRUCTURE. I AM PLEASED TO SAY WE HAVE FINALLY ENACTED THIS ELEVATION PROCEDURE. WE NOW HAVE DIVISION OF EXTRAMURAL ACTIVITIES AND THE DIRECTOR OF THAT DIVISION AND WEIRD DISEASE INNOVATION THERE ARE MANY WAYS TO SAY THIS ACRONYM AND I ENCOURAGE YOU TO SAY ALL OF THEM WHICHEVER YOU LIKE THE BEST I'M PLEASE TO SAY THAT PJ BROOKS IS ARE ACTING DIRECTOR AND WE ARE CONDUCTING A SEARCH FOR A DIRECTOR IN THIS RULES THAT WILL TAKE PLACE RELATIVELY SOON. >> AND THEN WERE ALWAYS PLEASED TO SEE IN THE LIMELIGHT. SO FROM THE ASSOCIATION OF AA AND SEE WITH THE 2022 INNOVATION TO BOLSTER TRUST AND SCIENCE AWARD WINNERS THREE LEADERS ARE RECOGNIZED RECEIVING FIRST PLACE AND ALSO THIRD-PLACE IN A TIE. SO WE ARE EXCITED TO SEE THAT ONCE AGAIN COMING TO COMMUNITY ENGAGEMENT. OF COURSE THERE IS MORE WORK TO DO BUT THESE ARE A FEW OF THEM WE'RE IN A GREAT POSITION TO CONTINUE TO DO BETTER WHEN IT COMES TO BOLSTERING COMMUNITY TRUST I'M EXCITED TO SEE STUDENTS AWARD WINNERS. IN ADDITION OF FRIDAY AFFORDS WERE GIVEN I WANT TO HIGHLIGHT TWO OF THE TRAINEES WITHIN THE PROGRAM. POSTDOC ON THE LEFT AND OUR PRE- DOCTORAL FELLOW. CONGRATULATIONS AND THEIR WORK TO BE RECOGNIZED WHAT THEY ARE DOING. LASTLY I WANT TO ROUND OUT THIS AWARD ANNOUNCEMENT WITH THE PUBLIC SERVICE AWARD 2022 FOR WORK AND CONSISTENTLY FOSTERING THERAPY THROUGH WORKING WITH THE GOVERNMENT AGENCIES WORKING TIRELESSLY IN THE SPACE AND THIS MEETING IS HAPPENING AS WE SPEAK. CONGRATULATIONS FOR RECEIVING THIS AWARD. MUCH-DESERVED. ALSO WHILE I AM I'M GENE AND CELL EDITING I MENTIONED THE PROGRAM WE ARE NOW IN PHASE NUMBER TWO AND ANNOUNCEMENTS ARE ON THE STREET I WANT YOU TO BE AWARE OF THESE ANNOUNCEMENTS TO DEVELOP NOVEL TECHNOLOGIES AND GENE TO BUILD FOUNDATIONS FOR MEDICAL APPLICATIONS FOR GENE EDITORS AND DELIVER RESOURCES SO THAT'S THE FOCUS OF THESE ACTIVITIES WITH THE S ECG PROGRAM AND WE ARE EXCITED HOW THIS WILL TURN OUT BECAUSE IT'S GETTING INTO THE CLINICAL SPACE WHERE WE REALLY NEED TO DO A BONE ON —- A BIG PUSH TO BRING THERAPIES TO ALL PEOPLE. I WANTED TO LET YOU KNOW ABOUT A COUPLE OF MEETINGS WE HAVE HELD. THIS IS COMING IN THE FUTURE JUNE 7 AND JUNE 8 HERE IS THE QR CODE THIS IS ON THREE D TISSUE MODELS AND HOSTED BY THE BILL AND MELINDA GATES FOUNDATION AND IT WILL PROVIDE HIGH-LEVEL PERSPECTIVES ON INFECTIOUS DISEASE AND THE POTENTIAL VALUE OF THREE D TISSUE MODELS AS WELL AS TOOLS FOR UNDERSTANDING AND MODELING THOSE INFECTIOUS DISEASES IT'S AN IMPORTANT MEETING AS THREE D TISSUE MODELS AND MICRO PHYSIOLOGICAL TAKES OVER THESE MEETINGS WILL BE MORE IMPORTANT WITH USEFULNESS AND THE APPLICABILITY TO RESEARCH. THIS MEETING WAS LAST MONTH I JUST WANT TO HIGHLIGHT BECAUSE WE HAVE HELPED TO PROVIDE SOME OF THE INFORMATION RELATED IN THE MEETING AND WE HAD ABOUT THOSE AWARDEES WHO PARTICIPATED IN THIS RESEARCH SESSION AND EXPLORING TRENDS AND THOSE STAKEHOLDERS SO THEY CAN HEAR ABOUT THEIR CONCERNS AND IDEAS AND AS WE THINK ABOUT THE INVESTMENT OF THE SCIENCE AND THE SOLUTIONS TO MOVE THE OPIOID EPIDEMIC AND THOSE PAIN RELATED DISEASES TO MOVE THOSE THINGS FORWARD. AND THAT WHAT HAS BEEN SUPPORTED WITH THAT VARIETY OF PRESENTATION I JUST WANT TO GIVE YOU AN UPDATE AND WITH THOSE OPPORTUNITIES THAT WE ARE DOING IN THAT SPACE. SO YESTERDAY SO THE STAKEHOLDER TEATIME THAT WAS VERY WELL ATTENDED AND TO TALK ABOUT HOW THOSE TO BRING THE COMMUNITIES TOGETHER OF HOW TO FLUSH THOSE GOALS OUT A LITTLE BIT MORE AND THAT IS SO IMPORTANT. AND VERY EXCITED ABOUT THE TURNOUT. >> TURNING TO THE BUDGET THIS IS CONFUSING BECAUSE OF THE TIMING HOW EVERYTHING HAPPENED. AS I MENTIONED ON MARCH 15 AND OF THE FIVE.2 PERCENT. ALSO THREE.1 PERCENT INCREASE THERE WAS A THREE.3 PERCENT INCREASE AT NIH AND ALSO PROVIDED $60 MILLION FOR THE NETWORK TO CONDUCT ITS BUSINESS SO THIS WAS ENACTED MARCH 15. AND WITH THE FY 23 BUDGET IS BECAUSE THROUGH THE CONTINUING RESOLUTIONS ARE FY 22 SO THE TIMING OF THE RELEASE AND THE PRESIDENT'S BUDGET WAS NEARLY IDENTICAL. IT WOULD HAVE TAKEN A LOT OF WORK TO GO BACK AND REDO THE ENTIRE BUDGET BASED ON THE APPROPRIATIONS WHEN YOU'RE FAMILIAR WITH THE PRESIDENTS BUDGET THIS IS THE INITIAL STAGE OF THE CONVERSATIONS I DO EXPECT THERE WILL BE MORE UPDATES GOING FORWARD BUT THAT IS THE WRINKLE BECAUSE IT DOES MAKE THE NUMBERS ARE DIFFERENT. AND THEN WITH THAT ENACTED BUDGET. BUT WHEN YOU LOOK AT THE PERCENT CHANGE IT'S NOT THAT FAR AWAY. AND JUSTIN THE PRESIDENTS BUDG BUDGET. WE WILL SEE HOW THAT PLAYS OUT AS WELL. THE CONGRESSIONAL HEARINGS ON THE NIH BUDGET AND THOSE WERE WELL RECEIVED SO SO THIS INCREASE OF 35.SEVEN BUT THIS IS REFERRING TO THE 5 BILLION INCREASE AND ALSO A 12 BILLION-DOLLAR EXPENDITURE FOR THE PANDEMIC PREPAREDNESS. AND IT WILL CONTINUE TO KEEP YOU POSTED. >> SO WITH THOSE ACTIONS A COUNSELOR TAKE IT ON WITH THOSE FUNDING OPPORTUNITIES THEY WITH THE FAMILIAR TO YOU WITH A RARE DISEASE AND HONESTY TSA PROGRAM. I ENCOURAGE YOU TO TAKE A LOOK AT THOSE AS WELL. >> THIS IS THE SAME FOR THE FUNDING OPPORTUNITY TO SEE A FRIDAY OF FUNDING OPPORTUNITIES AND THESE ARE THE ONES TO LEARN MORE OF THE BUSINESS TO GO ON THE QR CODE. A COUPLE OF PROGRAMS WE ASKED THEM TO PROVIDE THEIR OWN UPDATE TO YOU AND NOW THE OFFICE OF SPECIAL INITIATIVES AND THEN DAN FROM THE SPECIAL INITIATIVES OFFICE. I AM SUPER EXCITED THIS IS A CAPSULE THAT HAS WITH THE FIRST-EVER FDA DRUG APPROVAL AND TO THE THEIR PUTATIVE DEVELOPMENT BRANCH SO ANOTHER CONCEPT THIS IS A TWO-PHASE COMPETITION FROM THE PROGRAM AND LOOKING AT NATURAL LANGUAGE PROCESSING ABSTRACT LOOKING AT HOW THEY COULD BE CONCEPTS COULD BE PULLED OUT OF THE ABSTRACT THAT IS THE CONTEXT BY WHICH THEY APPEAR IN THE ABSTRACT. THAT IS ONE OF THE THINGS THEY COULD DO EFFECTIVELY SO THIS IS A RESULT OF RUNNING THE NATIONAL LANGUAGE PROCESSING SYSTEM AND THEN THOSE SCORES WOULD APPLY TO GENERATE WINNERS FOR THIS PARTICULAR CHALLENGE OVER 200 PARTICIPATING TEAMS AROUND THE WORLD AND THE HIGHEST SCORING TEAM IS REPRESENTED ON THE MAP HERE WE WILL BE REWARDING $100,000 IN PRIZES TO US CITIZENS AND PERMANENT RESIDENTS FOR THESE DIVERSE TEAMS. WE WILL HEAR MORE BECAUSE THERE IS NORMALLY HAVE TO APPROVE TO THE PROCESS SO STAY TUNED TO HEAR MORE ABOUT THOSE WE ARE GETTING CLOSER TO MAKE THIS IDEA A REALITY. THERE HAS BEEN OVERALL EXCITEMENT SO WE ARE ENCOURAGED BY THAT AND WE HOPE THAT YOU AR ARE. ANOTHER ACTIVITY WE ARE INVOLVED IN CONNECTED TO THE PROGRAM YOU'LL NOT NECESSARILY SEE THEM THAT THIS IS THE GENE THERAPY CONSORTIUM THAT YOU HEARD ABOUT AND TO PROVIDE A VERY BRIEF UPDATE WITH THAT BIOLOGY APPLICATIONS ON THE STREET AND WE ARE AWARDING FOR THOSE APPLICATIONS AND THEN ALSO CONDUCTING THIS PARTICULAR PROGRAM CBS AND IH AND IN ADDITION AND WITH THAT MANUFACTURING AREA AND THEN TO DEVELOP THOSE ATTRIBUTES AND THEN TO ENSURE WE HAVE A ROBUST MANUFACTURING PROCESS WITH THE TOXICOLOGY SIDE IN TERMS OF MANUFACTURING AND PRODUCTION OF THESE TYPES OF GENE THERAPIES AND THEN ULTIMATELY WE ARE FINALIZING THE DISEASE NOMINATION PROCESS AND WITH THOSE DISEASE NOMINATIONS FOR THE GENE THERAPY TRIALS AND A LITTLE OVER THOSE PROPOSALS THAT WE HAVE AN ANNOUNCEMENT WE EXPECT TO DO IN THE FALL I DO GIVE YOU AN UPDATE IN SEPTEMBER PROGRESS IS BEING MADE. WE HAVE HAD A VARIETY OF DISCUSSIONS WITH THOSE INTERWOVEN PLANS AND THEN TO HOLD A ROUNDTABLE BACK IN MARCH AND THIS WAS A VERY SMALL GROUP OF PEOPLE TO DISCUSS A VARIETY OF ACTIVITIES OF CHALLENGES AND OPPORTUNITIES AS THE FIELD TRAINS AND EDUCATES THE NEXT GENERATION IN THE SPACE EACH OF THE PARTICIPANTS WERE ASKED TO PROVIDE A ONE WORD RESPONSE BUT THEY THINK OF TRAINING AND EDUCATION AND THOSE ARE THE KEYWORDS HIGHLIGHTED IN THE BUBBLES ARE VERY OPTIMISTIC TONE HOW TRAINING EDUCATION IS BEING PERCEIVED. I KNOW JESSICA AND HER TEAM ARE UP TO THE CHALLENGE. IS DIFFERENT FROM THE ROUNDTABLE SHOWN HERE. AND THEN TO GENERATE EXCITEMENT AND WITH THIS DIGITAL BADGING PROGRAM. NOT JUST WITHIN TRANSLATIONAL SCIENCE TO BRING MORE ENGAGEMENT TO THAT IDEA AND THEN WITH THOSE EDUCATIONAL OPPORTUNITIES AND THERE ARE MORE OF THE BEING PLANNED. WITH OTHER SOCIAL MEDIA SITES AND THEN TO CREATE AWARENESS AROUND THAT. THIS IS ONE OF THE ACTIVITIES ALSO THE INTER- AGENCY FELLOWSHIP WE INITIATED THIS AS YOU REMEMBER LAST YEAR SO THESE ARE THE TWO INCOMING FELLOWS FOR THIS YEAR WANT TO WELCOME THEM BOTH. AND THEIR PARTICIPATION IN THE PROGRAM. THIS IS A SECOND CLASS TO GO THROUGH THIS AND WE CONTINUE TO SEE TOP-NOTCH FELLOWS FOR THIS PROGRAM. IT IS QUITE COMPETITIVE. SO WHAT I WANT TO POINT OUT IS JUSTICE BECAUSE THAT BIDEN ANNOUNCED THAT NIH HAS LICENSED 11 COVID-19 RESEARCH TOOLS AND EARLY-STAGE DIAGNOSTIC CANDIDATES TO THE PATENT POOL THROUGH THE WHO. AND THIS IS MEANT TO BENEFIT PEOPLE LIVING IN THE LOWER MIDDLE INCOME COUNTRIES AND THROUGH THE AND ID. SO LET'S GIVE A SHOUT OUT FOR THIS DEVELOPMENT FOR THIS EXCITING ACTIVITY TO TRANSLATE THESE TECHNOLOGIES. IN ADDITION TO NATIONAL SEE ON —- BNC THREE WE ARE PLEASED TO SEE DEPARTMENT OF THE SERVICES IT WAS CALLED OUT SPECIFICALLY IN THIS PLAN AS A TOOL TO ACCELERATE ADVANCEMENTS IN SCIENTISTS AND RESEARCH FOR ALL. WE ARE VERY EXCITED IT'S IN THE STRATEGIC PLAN SO WANT TO HIGHLIGHT FOR THAT AS WELL ALSO THERE'S BEEN A LOT OF ACTIVITY IN THE SPACE TOO MUCH TO RELAY ADEQUATELY THAT WE ARE ENHANCING THE DATATYPES COMING INTO THE PROGRAM WITH THE ARRANGEMENT THE CMS AND NOW TO MAKE SURE THEY ARE FORMATTED CORRECTLY WITH ACCESS BY THOSE USERS WE ALSO HAVE SOCIAL DETERMINANTS MORTALITY DATA AND CLINICAL DATA AS WELL AS VACCINE DATA A COUPLE OF STATES THAT ARE IN THAT AND C-3 SO WE HAVE A MUCH BIGGER PICTURE FOR THOSE PATIENTS AND THAT HELPS US TO ROUND OUT THE AVAILABLE INFORMATION TO HELP US UNDERSTAND MORE ABOUT THIS VIRUS AND HOW TO TREAT AND DIAGNOSE IT IS OUR EXCITING DEVELOPMENTS WE HAVE A RELATIONSHIP WITH THE RECOVER PROGRAM SUPPORTING SOME OF THESE NC3 ACTIVITIES. ON THE LEFT-HAND SIDE IT IS AN UPDATE OF THE STATUS OF THE DATA OF AN ERCOT WE HAVE CROSSED THE 5 MILLION MARK FOR COVID POSITIVE PATIENT WITHIN AND THREE C IT'S UNFORTUNATE WE STILL HAVE SOME WAY PEOPLE CONTRACTING THE VIRUS BUT AT THE SAME TIME ALLOWS US HAVE MORE DATA IT IS VERY HELPFUL IF YOU WANT TO LEARN ABOUT OTHER TYPES OF DATA SET FOR THE RECOVER PROGRAM SPECIFICALLY LOOKING AT LONG COVID THE ENTRY TEAM HAS IDENTIFIED OVER 200,000 ADULTS WITH LONG COVID AND MAYBE READY FOR THE STUDIES I WENT TO HIGHLIGHT THE WORK THEY HAVE DONE IN THIS PHASE RECENTLY PUBLISHED THIS WEEK WITH THE QR CODE ON THE LOWER RIGHT HAND SIDE IS THE FIVE-STEP ANALYSIS TO IDENTIFY LONG COVID ON POST PATIENT. THEY HAD TAKEN THE HR ON PATIENTS WHO HAVE BEEN DIAGNOSED WITH THE CODE FOR LONG COVID WE CAN USE THAT IDENTIFY PATIENTS AND THEN GO BACK TO FIND THE PATTERNS WITHIN THE STATIONS AND APPLY THAT TO THOSE HAVE NOT BEEN DIAGNOSED WITH LONG COVID PER SE BETTER IN THE DATABASE USING THESE PHENOTYPES THROUGH THE LEARNED PATTERNS THIS IS A NICE WAY TO IDENTIFY THE COLD WAR FOR A LONG COVID AS WELL AS OTHERS BUT IN ADDITION ONE OF THE KEY TAKEAWAYS AND THEN THOSE DIFFERENT PHENOTYPES IS IMPORTANT AND THEN CONDUCT CLINICAL TRIALS AND THAT COULD BE HELPFUL AND THAT THEY TAKE AWAY IS THAT PROCESS THAT IS USED TO HELP US IDENTIFY THESE CASES THEN THEY WILL BE RECRUITED TO PARTICULAR STUDIES SO THIS IS A BIG LEAP FOR N3C THAT QUITE A BIT OF RESULTS ARE REPORTED AS WELL LOOK AT VACCINE INFORMATION RELATED TO THOSE WHO HAVE LONG COVID COMPARED TO THOSE WHO DON'T AND USE THAT DATABASE LOOKING AT DIFFERENT DOSING OF THE VACCINES AND THE ANALYTICS AND USING THOSE CONTROLS THE VACCINATION BEFORE THE ACUTE INFECTION REDUCE THE LIKELIHOOD OF LONG COVID BY 18 PERCENT. THAT IS PRETTY ROBUST CONFIDENT SO IT STARTS TO DEMONSTRATE THAT THERE IS POWER BEHIND USING THE ELECTRONIC HEALTH RECORDS AS REAL-WORLD EVIDENCE TO PRIORITIZE HOW WE MIGHT TACKLE LONG COVID AS WELL AS OTHERS RELATED TO IT. THAT IS EXCITING AND FINALLY TWO MORE THINGS THEY ARE CONTINUING TO COLLECT DATA FOR THE DATA PORTAL OVER 10000 DATA POINTS IT IS REMARKABLE THE WORK IN A QR CODE. AND THOSE ARE VISIBLE AND THEN TO HAVE VERY DETAILED INFORMATION AND THEN LASTLY ROUNDING OUT THIS SECTION I WILL NOT TALK TOO MUCH ABOUT THE COVID CLINICAL TRIALS I MENTIONED IT BEFORE WE ARE IN A HOLDING PATTERN WE ARE EXPECTING RESULTS ANY MOMENT THAT PROBABLY THE NEXT COUPLE OF WEEKS YOU MAY HEAR ABOUT TOP LINE RESULTS FROM THE DIFFERENT CLINICAL TRIALS. I JUST WANTED TO REMIND YOU OF THAT. STAY TUNED. SO NEXT I WILL END PRESENTATION ON THE SECTION OF RESPONDING TO A QUESTION THAT WE PROVIDED A REPORT ON THE TRIENNIAL ON MONITORING ADHERENCE TO THE POLICY ON THE INCLUSION OF WOMEN AND MINORITIES IN CLINICAL RESEARCH. AS YOU MIGHT RECALL WE COULD DO MORE TO HELP ADDRESS WOMEN AND MINORITIES IN CLINICAL RESEARCH AND THE DATA IS DIFFICULT TO INTERPRET THAT IS SAFE TO SAY THAT WE CAN DO BETTER SO I REALLY WANTED TO FOCUS AND NEXT SEVERAL SLIDES HOW WE ARE ADDRESSING THIS PARTICULAR ACTIVITY WITHIN THE WORK THAT WE DO GOING FORWARD THAT GIVES ME A GOOD OPPORTUNITY OF THE THREE AUDACIOUS GOALS AND IN ADDITION TO ALL PEOPLE BEING AT THE CENTER STAGE THERE ARE THINGS THAT UNDERLIE WHAT CAN GO INTO HOW WE NEED TO ADDRESS THAT. SO THESE ARE TWO OF THE MAIN PROGRAMS THAT ADDRESS THIS AND THEN WHEN THEY GIVE THEIR UPDATE THEY WILL TOUCH UPON THESE EFFORTS SO THIS WILL BE A THEME THAT GOES THROUGHOUT THE PRESENTATION TODAY AND TOMORROW SO FOR THEIR RARE DISEASE SPACE THIS WILL NOT BE A SURPRISE TO YOU BUT THERE ARE HEALTH DISPARITIES THAT INTERSECT WITH THE CHALLENGES DISEASE PATIENTS THEY HAVE EXPERIENCED AND WE KNOW FOR EXAMPLE THAT PAIN MANAGEMENT IS A BIG ISSUE PEOPLE WITH SICKLE-CELL DISEASE. SO THOSE OF AFRICAN-AMERICAN DESCENT IT IS AN AMPLIFIED ISSUE FOR SICKLE-CELL PATIENTS WHO ARE TRYING TO RECEIVE PAIN MANAGEMENT. THAT ODYSSEY IS ANOTHER CHALLENGE. ESPECIALLY THOSE UNDERSERVED VOICES AND THEN TO OBTAIN A BETTER UNDERSTANDING WE LOOKED AT 22 PRIORS AND THE BASELINE AND THAT WE CAN INVOKE GOING FORWARD TO ADDRESS ANY DISPARITIES THAT WE HAVE AND ISSUES AROUND THAT INCLUSION THAT WE HAVE. SO RARE DISEASES AFFECT A VERY SMALL POPULATION WITH THE INDIVIDUAL DISEASE SO THAT IS MOSTLY UNKNOWN BUT THAT STILL MEANS WE NEED TO DO MORE TO FIND OUT HOW WE NEED TO MAKE SURE WE ARE INCLUDING THOSE MINORITIES AS WELL WITH THE INCLUSION OF THE WORK THAT WE DO. WITH VERSION FIVE IT OUT AS WE HAVE ADMINISTRATIVE SUPPLEMENTS AND ALSO PROMOTING SPECIAL INTEREST GROUPS AND WITH PATIENT ADVOCACY GROUPS. AND WITH THAT AMNESTY PROGRAM WE HOPE THAT ALLOWS US TO BRING THE INNOVATIONS TO BE APPLIED AS WELL. SO THIS TO BE A VARIETY OF WAYS TO INCREASE THESE ACTIVITIES FOR THE PROGRAM. IN ADDITION AND THEN TO MAKE THAT ACCESSIBLE AND THE ALSO MAKE SURE IT'S ENGLISH AND SPANISH AND I WANT TO EMPHASIZE THE WORD REVAMPING WE JUST STARTED SO WE ARE A BETA SITE LOOKING FOR INPUT FROM THE COMMUNITY FOR WHAT THEY WOULD LIKE TO SEE HER HOW THEY THINK AND LOOKING FOR THAT FEEDBACK SO THERE WILL BE MORE THINGS TO COME SO PLEASE STAY TUNED THIS WILL TAKE US NINE MONTHS BEFORE WE ARE COMPLETELY FINAL WITH THE WEBSITE BUT I THINK THESE CHANGES ARE IMPORTANT AND GET TO THE IDEA OF THAT ACCESSIBILITY FOR THE INFORMATION THAT IS DISPLAYED. >> VERY SIMILAR KINDS OF ACTIVITIES IN TERMS OF DOUBLING DOWN ON THE EFFORTS FOR DIVERSE POPULATIONS AND THINK HOW THEY HAVE TO BE REPRESENTATIVE OF THOSE AND HAVING THEM START TO LOOK FOR THOSE ACTIVITIES LOOKING AT THE RECRUITMENT INNOVATION CENTERS BUT THERE ARE A VARIETY OF PROGRAMS THAT ARE AN INCREDIBLE RESOURCE FOR ACTIVITIES AROUND COMMUNITY ENGAGEMENT AS WELL AS CULTURAL AMBASSADORS FOR THE WORKFORCE AS WELL I WENT TO HIGHLIGHT A FEW THINGS ON THE RIGHT-HAND SIDE THE COUNCILS OF FAITH AND USED ACROSS THE BOARD WITHIN THE COMMUNITY AND WE ALSO HAVE A LOT HEALTH TO CALL AND SPECIFICALLY THE UNIVERSITY OF SOUTH CAROLINA THEY ARE THE TELEHEALTH CENTER OF EXCELLENCE. WE DO HAVE A LOT OF THE LEADERSHIP HERE TO HELP US WITH THESE ACTIVITIES. >> IN ADDITION TO THAT WE ARE FINALIZING THIS ON OUR COMMUNITY ENGAGEMENT EFFORTS IN MANY OF THESE THAT I TALK ABOUT TODAY. IN ADDITION THE CTS A PROGRAM STEERING COMMITTEE HAS ASKED WE PULLED TOGETHER A DIVERSITY TASK FORCE TO MAKE RECOMMENDATIONS TO THE STEERING COMMITTEE ON FOCUSED EFFORTS AND IN TURN APRIL THAT PUBLICATION FROM THE TASK FORCE AND I REALLY RECOMMEND YOU TAKE A LOOK AT THIS PUBLICATION THEY PROVIDE A ROADMAP FOR SOME OF THESE ACTIVITIES AND IT TURNS OUT THE STEERING COMMITTEE HAS ELEVATED THE TASK FORCE TO AN ENTERPRISE COMMITTEE SO IT'S A CONTINUING ASPECT OF LEADERSHIP SO WE ARE EXCITED TO SEE THAT IS ALSO THE CALL FOR MANUSCRIPTS THROUGH J CTS TO FOCUS ON CLINICAL AND TRANSACTIONAL SCIENCE SO THAT CALL IS STILL UNDERWAY SO THIS IS SOMETHING ELSE THAT WILL HELP TO RAISE AWARENESS OF THE ACTIVITIES WITHIN THE PROGRAM TO PROVIDE THAT LEADERSHIP TO OTHERS IN THE COMMUNITY WITH THE RECRUITMENT INNOVATION CENTER I DON'T KNOW IF WE WILL EVER HAVE A REALLY GOOD WAY TO MEASURE A CREDIBLE BENEFIT OF THESE ACTIVITIES BUT I WANTED TO HIGHLIGHT A FEW OF THEM THE FASTER TOGETHER IS A PLATFORM DEDICATED TO IMPROVING THE REPRESENTATION OF RACIAL AND ETHNIC MINORITIES AND RESEARCH TO HELP TEACH AND TRAIN PEOPLE HOW TO DO THIS HELPS TO HIGHLIGHT PARTICULAR COMMUNITIES THAT DO THIS VERY WELL SO PEOPLE CAN REACH OUT TO THEM AND PARTNER WITH THEM MOVING FORWARD ALSO ROBUST ACTIVITY WITH THE METHODS TO ENCOURAGE DIVERSE GROUPS OF STAKEHOLDERS TO IMPLEMENT ALSO A COMMUNITY ADVISORY BOARD TO HELP PROVIDE INPUT AND RECOMMENDATIONS AND THERE ARE 12 MEMBERS ON THE BOARD REPRESENTING A VARIETY OF PEOPLE FOR THESE ACTIVITIES SO THIS IS ONE OF THE THINGS THAT HAS SHOWN TREMENDOUS BENEFIT AND NOW WE EVALUATE THIS MORE TO UNDERSTAND WITH THE BENEFIT LOOKS LIKE BECAUSE THAT RICK CONSULTS SO IT'S DIFFICULT TO OBTAIN THAT ENROLLMENT DATA BUT WE HOPE WE CAN FIGURE OUT WAYS TO ENHANCE THAT AND WE ARE WORKING WITH THE INCLUSION OFFICER AND THE VANDERBILT TEAM TO FIGURE OUT BEST METHODOLO METHODOLOGIES. I HOPE I CAN TALK MORE ABOUT THIS AND JUST A FEW MORE THINGS AND JUST WANT TO MENTION IT HERE BECAUSE THAT IS ONE OF THOSE THINGS TO FOCUS ON THE UNDERSERVED POPULATIONS AND IT ENSURES ALL AMERICANS HAVE ACCESS TO COVID-19 TESTING AND FOCUSES ON COMMUNITIES MOST AFFECTED BY THE PANDEMIC SO IT HAS BEEN A FANTASTIC PROGRAM FOR NIH AND THE CTS A IS A BIG PART OF IT. I ALSO WANT TO MENTION IN TERMS OF AN C-3 WE COLLECT DATA FROM HEALTH CENTERS ACROSS THE COUNTRY AND THAT INCLUDES FROM THE AMERICAN INDIAN AND ALASKA NATIVE IN THE DATABASE WE WANT TO MAKE SURE WE HAVE CONSULTATIONS WITH THE TRIBAL NATIONS TO UNDERSTAND HOW TO INCORPORATE THE VIEWS WE HAVE BEEN WORKING WITH THE WORKING WITH NIH WITH A VARIETY OF GROUPS ACROSS THE COUNTRY ON TRIBAL EFFORTS AND WE HAD TRIED TO LEARN FROM THEM —- PRACTICES SO TO DATE WE HAVE SCARE THE DATA FROM THE GROUPS AND AGGREGATE THEM UNTIL WE UNDERSTAND FROM THE TRIBAL NATION SO WE HAD A CONSULTATION WITH REPRESENTATIVES OF THOSE GROUPS AND THEY ARE PROVIDING US WRITTEN FEEDBACK AS WE SPEAK AND THAT WILL GIVE OUT IN THE SUMMER AND MAY BE DELAYED A LITTLE BIT AND RECEIVING SOME OF THE INPUT DUE TO UNFORESEEN CIRCUMSTANCES AND WE WILL GET THAT OUT AS SOON AS WE CAN BUT IT HAS BEEN A WORTHWHILE EXPERIENCE AND I'M EXCITED TO SEE >> THIS IS A PREAMBLE TO WHAT'S NEXT. MARIE BERNARD, NIH CHIEF OFFICERS OF WORKFORCE DIVERSITY WILL TELL YOU MORE ABOUT NIH EFFORTS TO PROMOTE DIVERSITY, EQUITY, AND INCLUSION IN BIOMEDICAL RESEARCH. I'LL LEAVE BROADER UPDATES TO HER. BUT I DID WANT TO MENTION ONE THING, THAT I MODERATED A SESSION THROUGH THE UNITE PROGRAM, FOR HEALTH CENTERS AND SYSTEMS TO UNDERSTAND WHAT THEY ARE THINKING AND WHAT INPUT THEY COULD PROVIDE US WITH, IN TERMS OF STRUCTURAL RACISM AND HOW WE CAN START TO MITIGATE THESE ISSUES. WE TALKED ABOUT CHALLENGES FOR PATHWAYS, RESEARCH OPPORTUNITIES. WE TALKED ABOUT OPPORTUNITIES FOR ADDRESSING HEALTH DISPARITIES AND HOW BEST TO GO ABOUT DOING THAT, SOME IDEAS THEY HAVE, AND SOME BARRIERS AND SOLUTIONS FOR NIH TO BE CONSIDERING. THINGS AROUND ACTIONS, THINGS AROUND POLICIES, THINGS ABOUT OUR OWN ENGAGEMENT STRATEGIES THAT COME OUT OF THE NIH. AND TRYING TO THINK ABOUT HOW WE CAN REVAMP THOSE TO, AGAIN, DO A BETTER JOB AND ENGAGING AND ENROLLING THESE UNDERSERVED MINORITIES INTO THE CLINICAL RESEARCH THAT WE DO. AND THEN LASTLY, I THINK THIS IS MY LAST SLIDE, I'M NOT SURE, BUT LASTLY, I'VE TALKED ABOUT THE WORK BEING DONE ON THE EXTRAMURAL SIDE FROM NCATS, BUT THERE'S AN EQUALLY ROBUST AMOUNT OF ACTIVITY THAT IS LOOKING INWARD AT NCATS. WITHIN OUR OWN STAFF AND WITHIN OUR BUSINESS PRACTICES. THESE EFFORTS DON'T MEAN MUCH UNLESS I AND OUR NCATS STAFF DON'T PRACTICE WHAT WE PREACH HERE. SO THAT'S ANOTHER IMPORTANT ASPECT THAT I WANTED TO RELAY THAT WE HAVE OUR OWN NCATS IDeA COMMITTEE AND HEALTH DISPARITIES ACTION PLAN TO FIND AND BENCH MARK SOME OF THESE STANDARDS FOR US TO BE THINKING ABOUT MOVING FORWARD. AND SO, THESE ARE SOME OF THE ACTIVITIES THAT WE'RE DOING. WE'VE DONE A SURVEY, AND WE'RE USING THAT SURVEY TO REALLY HELP US THEN FIGURE OUT HOW WE CAN BETTER INTEGRATE THESE IDEAS INTO OUR PRACTICES AND HOPEFULLY ELEVATE THAT EVEN MORE, FROM THE INTERNAL PERSPECTIVE. SO, OUR COLLECTIVE EFFORTS IN ADDRESSING INCLUSION, WOMEN AND MINORITIES IN CLINICAL RESEARCH, ARE REALLY TO DOUBLE DOWN ON RAISING AWARENESS ON THESE ONGOING EFFORTS. THERE ARE STILL GAPS IN THIS SPACE THAT WE NEED -- WE HAVE GOT TO CONTINUE TO ADDRESS TOGETHER. AND WE WANT TO HONE OUR AUDACIOUS GOAL AND BUILD METRICS I TALKED ABOUT HERE TODAY, INTO OUR NEXT STRATEGIC PLAN WHEN WE'RE READY TO DO THAT. AND ENSURE THAT THESE CHANGES AND ACTIVITIES REALLY DO TAKE ROOT. I THINK THAT'S MY LAST SLIDE. I THINK NOW WE CAN OPEN IT UP TO DISCUSSION. SO THANK YOU FOR YOUR ATTENTION. AND I WILL TURN IT OVER TO CLAIRE TO MODERATE THE DISCUSSION. >> OKAY. THANK YOU VERY MUCH, JONI. DO WE HAVE ANY QUESTIONS? PAULA? >> YES, THANK YOU. I REALIZE ARPA-H HAS BEEN KIND OF A FOOTBALL BEING PASSED BACK AND FORTH TO MULTIPLE PLACES. WHEN DO YOU THINK -- I KNOW READING THE TEA LEAVES -- A DECISION WILL BE MADE WHERE IT'S GOING TO LAND ESPECIALLY THOUGH IT SEEMS IT'S BEING FUNDENED AND WE DON'T HAVE A HOME FOR IT? >> I APOLOGIZE IF THAT'S CONFUSING BUT ARPA-H RIGHT NOW IS WITHIN THE NIH. SO ITS HOME IS HERE. AND, YOU KNOW, I THINK TO -- THERE'S NOT AN INTERIM DIRECTOR NAMED AS YET. SO I THINK THAT'S PART OF THE NEXT PROCESS THAT WILL BE UNDERWAY BEFORE WE KNOW MORE HOW THAT'S GOING TO HAPPEN. BUT UNTIL THEN, I THINK THERE ARE A VARIETY OF THINGS WE CAN DO. FOR EXAMPLE, WE HAVE A LOT OF SYSTEMS AND -- SYSTEMS AS IN OPERATIONAL SYSTEMS, HOW WE DO OUR GRANTS MANAGEMENT, HOW WE -- OUR DATABASE FOR THOSE GRANTS, IT'S CALLED THE ELECTRONIC RESEARCH ADMINISTRATION DATABASE, ERA. WE CAN START TO BUILD AN ALLOWANCE FOR ARPA-H TO USE THAT DATABASE. OTHER AGENCIES USE THAT SAME KIND OF DATABASE. THOSE ARE AN EXAMPLE OF SOME FEATURES WE'RE DOING NOW TO HELP START TO STAND IT UP. WHEN I MENTION IN THE NEWS YOU PROBABLY HEARD ABOUT SOME OTHER ACTIVITIES IN THIS SPACE, THOSE WERE IN DISCUSSION, AND SO UNTIL SOMETHING HAPPENS OR DOESN'T, I THINK RIGHT NOW WE'RE JUST MOVING FORWARD WITH THE IDEA THAT IT IS WITHIN THE NIH AND WE'RE CONTINUING TO PLAN FOR THAT ACCORDINGLY. >> THANKS. PAUL, YOU'RE NEXT. >> JONI, ON THE SAME TOPIC, ASSUMING IT STAYS IN THE NIH, YOU MENTIONED THAT OF COURSE THERE'S A GOAL TO KEEP IT COMPLEMENTARY, NOT COMPETITIVE, WITH OTHER INSTITUTES. IT SEEMS LIKE NCATS WOULD BE THE MOST VULNERABLE, IF YOU WILL, INSTITUTE OR MOST AT RISK FOR HAVING TO KIND OF WORK OUT THOSE COMPLEMENTARY DETAILS. WHAT'S THE PLAN AHEAD FOR THAT? >> YES, THANKS FOR THAT QUESTION, PAUL. YEAH, YOU'RE RIGHT. OFTEN TIMES WHEN ARPA-H IS DISCUSSED, NCATS IS ONE OF THE FIRST THINGS THAT GETS MENTIONED IN TERMS OF WHAT WE DO AT THE NIH ALREADY. I DO THINK THERE WILL BE A LOT OF COMPLEMENTARY APPROACHES. THE KEY WORDS AROUND ARPA-H ARE LARGELY AROUND ALZHEIMER'S DISEASE, CANCER, DIABETES, FOR EXAMPLE. AND AS YOU KNOW, THE BIG FOCUS FOR NCATS ISN'T ON ANY ONE DISEASE BUT IF THERE IS A BOLUS OF DISEASE IT'S CERTAINLY THE RARE DISEASES THAT WE FOCUS ON PRIMARILY. BUT WE HAVE ACTIVITIES IN ALL OF THIS SPACE. I SEE -- I DO SEE A VERY GOOD PATH TOWARDS WHAT THAT COMPLEMENTARY APPROACH WOULD LOOK LIKE. OF COURSE, I EXPECT AND HOPE THAT I WILL BE AT THE TABLE ONCE THE INTERIM DIRECTOR IS IDENTIFIED, AND THEN THE FINAL DIRECTOR, ONCE THAT INAUGURAL DIRECTOR IS APPOINTED TO HAVE THOSE KINDS OF DISCUSSIONS ABOUT WHERE WE CAN BE MOST COMPLEMENTARY AND I THINK THAT IS CERTAINLY A PERSPECTIVE THAT WE ALL SHARE AND HOLD AND PURSUE. >> THANK YOU. ONE MORE FOLLOW-UP. FROM THE VANTAGE POINT OF MAKING SURE THAT YOU ARE AT THE TABLE, OR NCATS LEADERSHIP IS AT THE TABLE, IS THERE ANYTHING THIS COUNCIL CAN DO TO HELP MAKE THAT HAPPEN? >> WELL, THANK YOU FOR THAT. AT THIS POINT, I THINK WE'RE REALLY IN THIS SORT OF WAIT AND SEE, AND REALLY UNDERSTANDING WHO THE -- YOU KNOW, WHEN WE'RE GOING TO START THINKING MORE SCIENTIFICALLY, HOW THAT MIGHT HAPPEN. SO, I WOULD SAY, YOU KNOW, CERTAINLY LET'S CONTINUE TO HAVE THE CONVERSATION, BUT FOR NOW I AM ANTICIPATING THAT THIS IS SOMETHING THAT WE -- I'M EXCITED ABOUT, AND I CAN SEE A VERY GOOD PATH FORWARD. AND WHETHER THAT'S A LETTER OF EXCITEMENT AND ENTHUSIASM OR MAYBE A HELP, A NUDGE IN THE DIRECTION, I'LL CERTAINLY KEEP YOU POSTED ON THAT. THANK YOU FOR OFFERING. >> OKAY. DO WE HAVE ANY OTHER QUESTIONS OR COMMENTS? >> CLAIRE, I SEE IN THE CHAT THAT KELLY MAY HAVE ASKED FOR A PAPER THAT I AM NOT -- I MAY HAVE MISSED THE QR CODE ON IT. SO, KELLY, CAN YOU TELL ME WHICH PAPER YOU WANTED AND I'LL MAKE SURE YOU GET IT. AND YOU'RE ON MUTE, BY THE WAY. >> THANK YOU. IT WAS THE ONE THAT YOU WERE REFERENCING RELATED TO DIVERSITY. IT DIDN'T HAVE A QR CODE ON IT. AND I WAS ACTUALLY WRITING NOTES BUT I WOULD LOVE TO READ IT. >> I WILL MAKE SURE -- I'LL GET THE LINK AND PUT IT IN THE CHAT. >> GREAT. THANK YOU VERY MUCH. >> YOU BET. >> OKAY. ANNIE, YOU HAVE A QUESTION. >> A COMMENT AND THEN A QUESTION. I WANTED TO COMMENT ON THE WORK BEING DONE ON THE GUARD PLATE. WE WENT THROUGH THAT QUICKLY. I WANT TO COMPLIMENT THE TEAM WORKING SO HARD ON THAT EFFORT, THAT'S REALLY CRITICAL OUTWARD-FACING TOOL FOR OUR RARE DISEASE COMMUNITY AND SO MANY OTHERS. AND I KNOW THAT WORKING ON, YOU KNOW, DR. RUTTER, YOU FOCUSED ON ACCESSIBILITY, I KNOW THE WORK DONE ON THE INTEROPERABILITY OF ICD CODES, MAKING SURE WE KEEP THAT UPDATED AND STATE OF THE ART. AND THERE'S A LOT OF COLLABORATION WITH PARTNER ORGANIZATIONS AND OTHER COMMUNITIES TO MAKE SURE THAT WHAT'S OFFERED THROUGH GUARD REMAINS REALLY THE MOST UPDATED RESOURCE IN THE U.S. FOR THE RARE DISEASE COMMUNITY. SO I WANT TO THANK NCATS FOR THE WORK YOU'RE INVESTING IN THAT. >> THANK YOU FOR THAT COMMENT, ANNIE. ERIC SAID -- I THINK HE'S SUPERMAN SOMETIMES. HE HAS WORK TIRELESSLY ON THIS AND HE HAS BEEN REACHING OUT TO A VARIETY OF DIFFERENT GROUPS AND INDIVIDUALS, TO HELP GET INPUT AND FEEDBACK ON THE WORK THAT WE'RE TRYING DO, IN TERMS OF HARMONIZATION PROCESS AND IN TERMS OF DATASETS THAT CAN BE INCLUDED ON GUARD AND IT HAS BEEN VERY, VERY, VERY HELPFUL AND FRUITFUL IN TERMS OF HELPING US NAVIGATE AND UPGRADE OUR SITE. AND I DO HOPE IT'S HELPFUL. I SHOULD ALSO POINT OUT TOO THAT ANNIE, AT EVERY LIFE, THERE ARE WONDERFUL RESOURCES. ONE OF THE BEST RESOURCES ON THE I.C. CODES OUT THERE. AND SO IF THERE ARE WAYS THAT WE CAN ALSO MAKE SURE THAT WE HAVE CONNECTIONS TO OTHER RESOURCES THAT CAN COMPLEMENT OR SUPPLEMENT ACTIVITIES WE HAVE OR INFORMATION THAT WE HAVE, THAT'S ONE OF THE OTHER THINGS THAT WE'RE LOOKING TO DO. HOPEFULLY WE WILL HAVE A ONE-STOP SHOP THAT MAY NOT BE EVERYTHING LOCATED ON OUR SITE BUT CAN POINT TO WHERE OTHER INFORMATION IS LOCATED SO RARE DISEASE PATIENTS DON'T HAVE TO WORK HARD TO FIND THE INFORMATION THAT THEY NEED. AND I SEE P.J. P.J. MAY WANT TO SPEAK UP AS WELL ON THIS TOPIC. >> YEAH, ANNIE, THANKS YOU FOR THE COMMENTS. CERTAINLY ERIC DOES A GREAT JOB. BUT I ALSO JUST WANT TO BE SURE WE ACKNOWLEDGE THAT IT'S A REAL TEAM EFFORT, AND THE NCATS INFORMATICS PEOPLE AND MANY OTHERS INVOLVED IN THIS TRANSITION AT GUARD 2.0, WHICH WE'VE BEEN VERY HAPPY WITH. >> THANK YOU, P.J. OKAY. ANY OTHER QUESTIONS OR COMMENTS? >> I LOVED THE QR CODES. >> THANK YOU. OH, YES. YES! FEEDBACK, YOU LIKE THOSE? OKAY. I ADDED THEM IF THE LAST MINUTE, IF I'M BEING HONEST. SO I MAY HAVE MISSED -- I APOLOGIZE FOR ANY LINKS I DID MISS. AND I'M HAPPY TO -- I DON'T THINK -- NEXT TIME I'LL BE SURE TO DO IT AHEAD OF TIME. >> THE DINOSAUR IS CUTE. >> YOU KNOW, I DON'T KNOW WHERE THAT DINOSAUR COMES FROM BUT I THINK IT'S PRETTY CUTE TOO. THE TECHNOLOGY IS NOT DINOSAUR. IT'S VERY CURRENT. ALL RIGHT. WELL, YOU KNOW, IF THERE ARE NO OTHER COMMENTS, WE MAY WANT TO TAKE A QUICK BREAK BEFORE THE NEXT SPEAKER. ANNA, WHAT IS YOUR SENSE? >> YES, I THINK WE CAN TAKE A 10-MINUTE BREAK. >> OKAY. >> WE'LL START AGAIN AT 2:20 OR SO. OUR NEXT TALK IS SUPPOSED TO START AT 2:30. WE MAY BE ABLE TO START IT A LITTLE BIT SOONER. 2:10 TO 2:20. >> THANK YOU. WE'LL SEE YOU IN ABOUT TEN MINUTES. AND THANK YOU FOR YOUR INPUT. IT IS MY PLEASURE TO INTRODUCE TO YOU DR. MARIE BERNARD, SHE'S CHIEF OFFICER FOR SCIENTIFIC WORKFORCE DIVERSITY, ALSO KNOWN AS COSWD, AND WILL LEAD INTRAMURALLY AND EXTRAMURALLY AND CO-LEADS THE NIH'S NEWLY ANNOUNCED UNITE INITIATIVE TO END STRUCTURAL RACISM. PRIOR TO JOINING COSWD, SHE WAS DEPUTY DIRECTOR OF THE NATIONAL INSTITUTE ON AGING, AND SHE SERVED AS PRINCIPAL ADVISER TO NIA DIRECTOR. SHE'S LED A VARIETY OF ACTIVITIES WITHIN THE AGING COMMUNITY BUT ALSO IN THIS SPACE OF NIH-WIDE INCLUSION, GOVERNANCE COMMITTEE, THE WOMEN OF COLOR COMMITTEE OF THE NIH-WIDE WORKING GROUP ON WOMEN IN BIOMEDICAL CAREERS. SHE IS ALSO IN THE NATIONAL LEADERSHIP ON GERIATRIC RESEARCH, TEACHING IN CLINICAL PRACTICE, WIDELY RECOGNIZED IN HER ACTIVITIES. PRIOR TO JOINING NIH WAS AN ENDOWED PROFESSOR AND FOUNDING CHAIRMAN OF THE DONALD W. REYNOLDS DEPARTMENT OF GERIATRIC MEDICINE, UNIVERSITY OF OKLAHOMA, COLLEGE OF MEDICINE. SHE WAS ASSOCIATE CHIEF OF STAFF FOR GERIATRICS AND EXTENDED CARE AT THE OKLAHOMA CITY VETERANS AFFAIRS AND MEDICAL CENTER, AND SHE LECTURED ON TOPICS INCLUDING NUTRITION AND FUNCTION IN OLDER ADULTS WITH PARTICULAR FOCUS ON UNDERREPRESENTED MINORITY POPULATION, UNDERGRADUATE OF BRYN MAWR COLLEGE, M.D. FROM UNIVERSITY OF PENNSYLVANIA, TRAINED IN INTERNAL MEDICINE, CHIEF MEDICAL RESIDENT, IT'S OF A DELIGHT TO HAVE HER TO TALK ABOUT COMBATING STRUCTURAL RACISM. DR. BERNARD? >> THANK YOU SO MUCH, DR. RUTTER. HOPEFULLY YOU CAN ALL NOW SEE MY OPENING SLIDE. YES? >> YES. >> OKAY. THANK YOU. SO, AS DR. RUTTER SAID, I HAVE THE PRIVILEGE OF SERVING IN TWO LEADERSHIP ROLES AT NIH. I'D LIKE TO SHOW MY HATS, THE HAT OF COSWD, AS MENTIONED, I WEAR THE HAT OF CO-LEAD OF NIH UNITE INITIATIVE TO END STRUCTURAL RACISM. AND BOTH OF THEM ARE FOCUSED ON ASSURING WE TAKE ADVANTAGE OF FULL RANGE OF TALENT THAT'S AVAILABLE OUT THERE. I HAVE A THIRD ROLE, NO HAT YET, CO-LEAD OF THE NIH DIVERSITY, EQUITY, AND INCLUSION AND ACCESSIBILITY STRATEGIC PLAN. LET ME TELL YOU WHY THESE ACTIVITIES ARE NECESSARY. I DON'T REALLY NEED TO TELL YOU. I THINK YOU ALREADY KNOW, THAT THE SORTS OF PROBLEMS THAT WE'RE TRYING TO ADDRESS IN BIOMEDICINE ARE LIKE TRYING TO DESCRIBE AN ELEPHANT SIGHTLESSLY. WE NEED TO COME COME BASED ON VIEWPOINTS, DISCIPLINES, EXPERIENCE, GEOGRAPHY, OFTEN RELATED TO DEMOGRAPHIC CHARACTERISTICS, AS WE TALK ABOUT COMMENTARY FROM LAST NOVEMBER. LET ME TALK AT A HIGH LEVEL ABOUT WHAT'S HAPPENING WITH THE DEIA STRATEGIC PLAN, DIVERSITY, EQUITY, AND INCLUSION AND ACCESSIBILITY. THINGS GOING ON WITHIN MY OFFICE, AND NIH UNITE INITIATIVE. THE DEIA STRATEGIC PLAN IS COMING ABOUT AS A RESULT OF TWO MANDATES ONE FROM LEGISLATION THAT LED TO FISCAL YEAR 21 BUDGET FOR NIH THAT CALLED FOR A DIVERSITY, EQUITY, AND INCLUSION STRATEGIC PLAN, INCLUDING SHORT- AND LONG-TERM GOALS TO ADDRESS RACIAL, ETHNIC, GENDER DISPARITIES AT NIH AS WELL AS TO IDENTIFY AND ADDRESS BARRIERS TO ACCESS, IN ACCESS TO NIH FUNDING BY INVESTIGATORS, RESEARCH IN HEALTH DISPARITIES. AS WRITTEN, THE RECORD LANGUAGE CALLED FOR THAT PLAN WITHIN 180 DAYS, IN LAW DECEMBER OF 2020. WE, NIH, WENT TO THE LEGISLATORS AND POINTED OUT IT WAS A LOT THAT WAS CHANGING AROUND THAT TIME AND IT WOULD BE MUCH BETTER PRODUCT IF WE HAD A LONGER TIMELINE FOR DEVELOPING THAT PLAN, AND WERE ABLE TO NEGOTIATE THE PLAN WOULD BE DELIVERED LATE SPRING, EARLY SUMMER OF 2021, RIGHT AROUND THIS TIME. WE HAVE IN THE INTERIM BEEN GIVING QUARTERLY UPDATES TO WHAT'S CALENDAR THE FOUR CORNERS. SO LEADS IN THE HOUSE AND SENATE FOR ALLOCATION AND APPROPRIATION. AFTER DECEMBER 20 OF 2020, WE HAD A NEW ADMINISTRATION COME IN, ONE OF THE FIRST EXECUTIVE ORDERS FROM THE BIDEN ADMINISTRATION IS EXECUTIVE ORDER 14035, CALLS FOR DIVERSITY, EQUITY, INCLUSION, AND ACCESSIBILITY PLAN FOR FEDERAL WORKFORCE. WE'VE COMBINED THE TWO GOALS IN WHAT IS BEING DEVELOPED. THERE WAS A GOVERNMENT-WIDE STRATEGIC PLAN THAT GOT ISSUED NOVEMBER 23, A ROADMAP TO IMPLEMENTING THIS EXECUTIVE ORDER. IT CALLED FOR AGENCIES LIKE HHS TO DEVELOP A PLAN BY MARCH OF THIS YEAR, AND NIH AS AN OPERATING GIVING UNDER HHS IS, AS I SAID, LOOKING TO GET OUR PLAN. WE HOPE TO HAVE IT COMPLETED BY THE END OF JUNE AND PUBLISHED MAYBE MID-JULY, AN UPDATE ON THAT JUNE 10 AT THE ADVISORY COMMITTEE TO THE DIRECTOR MEETING. WHAT WE SEE IS THAT THIS SHOULD BE SOMETHING THAT HAS BROAD VISION AND ASPIRATIONS FOR ALL OF NIH, VERY BROAD SCOPE TO IT, THUS WE HAVE MORE THAN 100 STAFF MEMBERS ACROSS NIH WORKING ON IT. WE'VE SPENT A LOT OF TIME ARTICULATING NIH DEFINITIONS WITH DIVERSITY, EQUITY, AND INCLUSION AND ACCESSIBILITY, LANGUAGE MATTERS. WE ARE DEVELOPING EXAMPLES OF ACCOMPLISHMENTS, CONVEYING PRIORITIES FOR THE NEXT FIVE YEARS, SAMPLE ACTIVITIES, IMPORTANTLY WE NEED TO PROVIDE ACCOUNTABILITY, SO WE'RE LOOKING AT MEASURES OF PROGRESS, AND WE GATHERED INTERNAL INPUT. WE'VE GOTTEN EXTERNAL INPUT, PARTICULARLY FROM RFI. AND THAT'S THE SCOPE OF THE PLAN, TO ARTICULATE NIH'S VISION FOR STRENGTHENING DIVERSITY, EQUITY, AND INCLUSION AND ACCESSIBILITY, TO CAPTURE ACTIVITIES THE NIH WORKFORCE WILL UNDERTAKE TO MEET THE VISION OF THE STRATEGIC PLAN, AND TO HARMONIZE THIS, NIH-WIDE STRATEGIC PLAN FLAME WORK, ORGANIZED AROUND THREE KEY AREAS, ONE IN THE WORKFORCE, DIFFERENT FROM YOUR STANDARD NIH PLAN. STANDARD NIH PLAN YOU HAVE RESEARCH FIRST AND THEN OTHER ISSUES. BUT BECAUSE THIS IS VERY PEOPLE-FOCUSED WE THOUGHT THIS SHOULD BE THE FIRST OBJECTIVE, NIH WORKFORCE, AND THEN WORKFORCE AT INSTITUTIONS SUPPORTED BY NIH. WE HAVE AS A SECOND OBJECTIVE TO GROW AND SUSTAIN DEIA THROUGH STRUCTURAL AND CULTURAL CHANGE, FAMILIAR STEWARDSHIPS, PUBLIC/PRIVATE PARTNERSHIPS AND ENGAGEMENTS, ACCOUNTABILITY AND CONFIDENCE, MANAGEMENT AND OPERATIONS. THIRD, RESEARCH OBJECTIVE, ON THE WORKFORCE, HEALTH RESEARCH. WE HOPE THAT YOU'VE HAD A CHANCE TO CONTRIBUTE TO THE RFI, IF NOT TUNE IN TO THE DIRECTOR MEETING WITH ANOTHER PUBLIC DISCUSSION OF THIS, AND HOPEFULLY WE WILL PUBLISH SOON. THEN FROM THE COSWD PERSPECTIVE WE'VE BEEN WORKING HARD ON DEVELOPING OUR OWN STRATEGIC PLAN, AND IN FACT IT WAS RELEASED AT THE BEGINNING OF MARCH OF THIS YEAR. IT OUTLINES WHAT OUR VISION IS FOR 22 THROUGH 26. WE HAVE A VISION OF ENABLING NIH AND NIH-FUNDED INSTITUTIONS TO BENEFIT FROM THE NATION'S FULL RANGE OF TALENT, BECAUSE WE KNOW THAT THERE'S A LOT OF TALENT OUT THERE. AND OUR GOAL IN DOING SO IS NOT ONLY TO ADDRESS EQUITY AND JUSTICE, BUT TO FOSTER CREATIVITY AND INNOVATION IN SCIENCE. I WILL BE SHARE SOME DATA RELEVANT TO THAT. OUR MISSION IS TO BE THE NIH THOUGHT LEADER IN THE SCIENTIFIC WORKFORCE DIVERSITY, USING EVIDENCE-BASED APPROACHES TO CATALYZE CULTURES OF INCLUSIVE EXCELLENCE. HOW DO WE ENVISION DOING THIS? BY BUILDING THE EVIDENCE, USING NIH AS A TEST BED FOR MANY INTERVENTIONS, BY DISSEMINATING EVIDENCE ACROSS THE BIOMEDICAL RESEARCH ECOSYSTEM, AND BY ACTING ON THE EVIDENCE BY ADVANCING INTEGRATIVE INSTITUTION-WIDE SYSTEMS. WE'RE THINKING BROADLY ABOUT DIVERSITY. AKIN TO THE NIH NOTICE OF INTEREST AND DIVERSITY FROM 2019. LET ME EMPHASIZE THAT THE CATEGORIES HERE ARE EXAMPLES, NOT AN EXHAUSTIVE LISTING OF GROUPS UNDERREPRESENTED IN THE BIOMEDICAL SCIENCES BUT COMES DIRECTLY FROM THAT NOTICE, FROM 2019, WHICH GIVES AS EXAMPLES INDIVIDUALS FROM RACIAL AND ETHNIC GROUPS SHOWN BY THE NATIONAL SCIENCE FOUNDATION TO BE UNDERREPRESENTED IN HEALTH-RELATED SCIENCES ON A NATIONAL BASIS. INDIVIDUALS WITH DISABILITIES. INDIVIDUALS FROM A DISADVANTAGED BACKGROUND. IF YOU LOOKED, YOU KNOW THERE ARE MULTIPLE CATEGORIES BY WHICH ONE COULD BE CONSIDERED DISADVANTAGED, YOU HAVE TO HAVE TWO OR MORE OF THEM, LIKE HAVING BEEN ON A SCHOOL LUNCH PROGRAM, RURAL BACKGROUND, THAT SORT. WOMEN AT GRADUATE LEVEL AND BEYOND IN SCIENTIFIC FIELDS. I SHARE THIS WITH YOU TO DEMONSTRATE WE'RE THINKING BROADLY, WHEREAS THE UNITE INITIATIVE IS VERY FOCUSED ON RACIAL AND ETHNIC EQUITY. SO WE'RE THINKING BROADLY ABOUT ALL OF THOSE DIFFERENT GROUPS THAT NEED TO BE AT THE TABLE TO ADVANCE SCIENCE HERE. SO, SOME EXAMPLES OF THINGS THAT WE'RE DOING. WE'VE LAUNCHED JUST THIS MARCH SOMETHING CALLED THE 21ST CENTURY SCHOLARS PROGRAM. THE GOAL HERE IS TO DEVELOP AN INCLUSIVE CULTURE BY HAVING A COHORT OF EXTRAMURAL PROGRAM STAFF WHO HAVE BUILT A COMMUNITY WHO HAVE GOTTEN EXTRA MENTORING, WHO HAD ENHANCEMENT OF THEIR SKILLS, TO BECOME BASICALLY DEIA AMBASSADORS, MODELED AFTER THE DISTINGUISHED SCHOLARS PROGRAM AT NIH THAT'S BEEN SUCCESSFUL INCREASING DIVERSITY OF TENURE TRACK SCIENTISTS IN INTRAMURAL PROGRAM. HERE THE VISION IS THAT, YES, IT MAY HELP INCREASE DIVERSITY OF EXTRAMURAL PROGRAM STAFF, BUT MAY ALSO TRANSLATE INTO DIFFERENCES IN WAY FUNDING OPPORTUNITY ANNOUNCEMENTS ARE WRITTEN IN THE FUTURE, OR WHO GETS INVITED TO SCIENTIFIC MEETINGS OR WHO ENDS UP BEING PICKED UP ON DISCRETIONARY PAY BY THE I.C.s. THE FIRST GROUP WE HAVE OUR TEST -- OUR PILOT GROUP THAT'S GOING ON RIGHT NOW, 13 PROGRAM PARTICIPANTS, 6 MENTORS. THEY ARE BUILDING A SELF-REINFORCING CULTURE THAT FOCUSES ON THE SCIENCE OF SCIENTIFIC WORKFORCE DIVERSITY. AND THEY MEET ONCE A MONTH. THEY GET TO HEAR FROM EXPERTS IN DEIA. THEY DO A JOURNAL CLUB LOOKING AT LITERATURE. THEY HAVE JUST GOTTEN LAUNCHED IN DOING WHAT WE CALL COSWD CHALLENGE, A PROJECT TO ADVANCE DEIA PRINCIPLES HERE AT NIH. AND WE WILL SEE WHETHER THIS IS SUCCESSFUL, COME SEPTEMBER WHEN THE PILOT PROJECT IS DONE WE DEEM IT TO BE SUCCESSFUL, THUS FAR LOOKS LIKE IT'S DOING WELL. WE'LL REPEAT IT ON ANNUAL BASIS FOR FOUR OR FIVE YEARS WITH THE HOPE WE'LL HAVE A CRITICAL MASS OF INDIVIDUALS WITH A RIPPLE EFFECT ACROSS I.C.s AND NIH. IN TERMS OF DISSEMINATING EVIDENCE WE STARTED THE SCIENTIFIC WORKFORCE DIVERSITY SEMINAR SERIES THIS PAST FALL. ON TUESDAY OF THIS WEEK, WE HAD THE FINAL OFFERING OF THE 21-22 SEASON, HOW DOES DIVERSITY IMPACT SCIENCE. WE WERE EXCITED. WE HAD MORE THAN 500 PEOPLE WHO ACTIVELY ATTENDED THE SESSION. WE HAD OUTSTANDING SPEAKERS, DR. ISLER GAVE US OPENING REMARKS. DOCTORS SMITH-D.O.E.RR, FREEMAN, KUAN AND SANTANGELO WITH THE IMPACT OF DIVERSITY ON SCIENCE. DR. TILGHMAN GAVE CONCLUDING REMARKS, SUCH RICHNESS OF THOUGHT WE DIDN'T GET TO DELVE IN GREAT DEPTH INTO ALL OF THE QUESTIONS PUT FORWARD BY THE PEOPLE PARTICIPATING BUT WE GOT TO TALK AT THE TOP LEVEL, THE WAY WE WOULD HAVE LIKED ABOUT THEMES AND TAKEAWAY POINTS, WHAT ELSE NEEDS TO BE DONE IN THE RESEARCH IN THIS AREA. SO IT WAS REALLY STIMULATING. IF YOU DIDN'T GET A CHANCE TO PARTICIPATE, THE VIDEOTAPE AND POWERPOINT PRESENTATIONS WILL BE ON OUR WEBSITE, IN THE NEXT SEVERAL DAYS. THERE WILL BE MEETING PROCEEDINGS THAT WILL BE POSTED SOMETIME THE NEXT MANY WEEKS. SO DO TAKE ADVANTAGE OF IT. WE WILL BE RESUMING THIS SEMINAR SERIES NEXT SEPTEMBER. ACTING ON THE EVIDENCE, IT'S VERY CLEAR THAT MENTORING IS IMPORTANT IN FOSTERING CAREERS OF GROUPS THAT DON'T NECESSARILY HAVE BUILT-IN SUPPORT NETWORKS. WOMEN. INDIVIDUALS FROM UNDERREPRESENTED RACIAL AND ETHNIC GROUPS. WHAT WE DID FOR THIS FISCAL YEAR IS TO DEVELOP AN ADMINISTRATIVE SUPPLEMENT OPPORTUNITY FOR DEIA MENTORING, A SUPPLEMENT ALREADY FUNDED RESEARCH, WE OPENED TO MULTIPLE MECHANISMS, AND WE GOT A THOUSAND INQUIRIES. WE HAVE ALL SORTS OF R01 RECIPIENTS TRYING TO CONVINCE US THEY QUALIFIED BUT HONESTLY YOU EITHER HAD TO HAVE A MENTORING OBJECTIVE IN YOUR R01 THAT WAS REVIEWED IN THE PEER PANEL, OR YOU HAD TO HAVE A PLAN TO ENHANCE DIVERSE PERSPECTIVE. THERE ARE VERY FEW FOAs THAT CALL FOR THAT CURRENTLY, OR HAVE AN ACTIVE DIVERSITY SUPPLEMENT ASSOCIATED WITH R01. LESS THAN 5% OF R01S HAVE DIVERSITY SUPPLEMENTS. NONETHELESS WE GOT A ROBUST RESPONSE, I.C.s ARE REVIEWING THESE AND WILL BE SENDING US TOP FIVE RECOMMENDATIONS. WE WILL PRIORITIZE FUNDING MEANT MENTORS WHO WILL ENHANCE, BUT WE'RE HOPEFUL SOME I.C.s WILL PICK THIS UP. ADDITIONALLY WE SEE THIS IS RECOGNIZING AN UNMET NEED AND ENVISION DOING IT AT LEAST AGAIN NEXT YEAR. FROM THE NIH UNITE PERSPECTIVE, NIH UNITE WAS DEVELOPED WITH THE VERY AMBITIOUS GOAL OF ENDING STRUCTURAL RACISM GENERATED BY THE EVENTS OF 2020 WHERE WE SAW DISPROPORTIONATELY MORBIDITY AND MORTALITY, FOLLOWED WITH RACIAL VIOLENCE, VIDEOTAPED MURDER OF GEORGE FLOYD, JUST AFTER IT WAS UNVEILED, THE KILLING OF SIX ASIAN WOMEN IN GEORGIA. WE'VE SEEN MORE IN THE WAY OF RACIAL VIOLENCE IN THE LAST MANY DAYS. NIH UNITE WAS UNVEILED TO ADDRESS WHAT WE COULD WITHIN THE BIOMEDICAL ECOSYSTEM FOR RACIAL AND ETHNIC EQUITY. EACH OF THE LETTERS OF UNITE REPRESENTS COMMITTEE THAT'S INTERACTING WORK STREAMS FOR THIS. ANOTHER WAY IS ENVISIONING THE WAY THAT UNITE IS FOCUSED, SHOWN IN THIS GRAPHIC, AGAIN FROM THE SAME NATURE MEDICINE COMMENTARY, A FOCUS ON HEMATOPOIESIS, -- ON HEALTH DISPARITIES, AS WE REQUEST INSTITUTIONS TO PAY ATTENTION TO HOW THEIR HOUSE IS ORDERED AS WELL. HIGHLIGHTS, AGAIN, 50,000-FOOT LEVEL, IN TERMS OF HEALTH DISPARITIES, MINORITY AREA, WE'RE COGNIZANT OF DATA THAT SHOWS DISPARITIES IN LONGEVITY, FOR INSTANCE. THESE ARE DATA THAT SHOWS LIFE EXPECTANCY FROM 1980 THROUGH TO 2020 FOR AFRICAN AMERICANS AND BLACKS, NON-HISPANIC WHITES, LATINOS, EVERYBODY HAD A DECREASE IN LONGEVITY WITH THE ONSET OF THE COVID PANDEMIC. BUT WE KNOW THAT THERE ARE PERSISTENT DISADVANTAGES FOR AFRICAN AMERICANS AND BLACKS RELATIVE TO WHITES, AND THERE HAVE BEEN PERSISTENT ADVANTAGES FOR LATINOS AND HISPANIC, RELATIVE TO WHITES, BUT THE WAY THIS HAS DROPPED WITH THE COVID PANDEMIC. WHAT ARE WE DOING? ONE OF THE FIRST THINGS ANNOUNCED WAS COMMON FUND TRANSFORMATIVE RESEARCH TO ADDRESS HEALTH DISPARITIES AND ADVANCE HEALTH EQUITY OPPORTUNITY, AND AT THE END OF FISCAL YEAR 21 COMMITTED UP TO $58 MILLION OVER THE COURSE OF FIVE YEARS FOR 11 PROJECTS ANNOUNCED OFFICIALLY IN OCTOBER. SIX OF THOSE WERE LOOKING AT THIS TOPIC IN GENERAL. FIVE OF THEM WERE LOOKING AT THIS TOPIC OF MINORITY-SERVING INSTITUTIONS, AND THERE'S BEEN FISCAL YEAR 22 COMPETITION THAT'S BEEN PROVIDED AS WELL. LOOKING FORWARD FISCAL YEAR 23, AS A RESULT OF UNITE INITIATIVE, CONCEPT OF A COMMUNITY PARTNERSHIP TO ADVANCE SCIENCE FOR SOCIETY HAS BEEN SUCCESSFULLY BROUGHT FORWARD TO THE COUNCIL OF COUNCILS. YOU CAN SEE MULTIPLE INSTITUTES, CENTERS, OFFICES ARE PART OF THIS. WHAT IS ENVISIONED IS COMMUNITY-DRIVEN HEALTH EQUITY STRUCTURAL INTERVENTIONS. IN OTHER WORDS, LOCAL HEALTH EQUITY RESEARCH ASSEMBLIES WHERE YOU HAVE PEOPLE ON THE GROUND SAYING WHAT IT IS THEY NEED TO IMPROVE THE HEALTH OF THEIR COMMUNITIES. COORDINATION CENTER WORKS WITH NATIONAL HEALTH EQUITY RESEARCH ASSEMBLY. AGAIN, COMMUNITY PEOPLE EXPRESSING WHAT THE NEEDS ARE. AND THEN HUB AND SPOKE ARRANGEMENT OF HEALTH EQUITY RESEARCH HUBS WITH SCIENTIFIC SUPPORT AND PARTNERSHIPS. VERY EXCITING CONCEPT. INTENT TO COMMIT 23 TO $52 MILLION PER YEAR OVER THE COURSE OF TEN YEARS WITH THIS BEING A FISCAL YEAR 23 INITIATIVE. IF YOU WANT TO HEAR MORE YOU CAN GO TO THE VIDEOCAST, JANUARY 27 VIDEOCAST OF THE COUNCIL OF COUNCILS PRESENTATION ON THE TOPIC. A HIGHLIGHT IN TERMS OF WHAT'S GOING ON IN THE INTERNAL WORKFORCE PERSPECTIVE, SOMETHING THAT YOU HOME WILL SEE NEXT COUNCIL SESSION, IF COUNCIL MEETINGS ARE IN PERSON, IS SOMETHING THAT WE CALL THE POWER OF INCLUSIVE WORKFORCE RECOGNITION PROJECT. THIS IS THE BRAINCHILD OF DR. JACKSON, DEPICTED HERE SECOND FROM THE LEFT ON THE BOTTOM ROW. SHE'S AN INTRAMURAL TENURE TRACK SCIENTIST AT NIH, AND AS VERY ELOQUENTLY STATED IN HER OP ED RECENTLY PUBLISHED, SHE TALKS ABOUT WALKING THE HALLS OF NIH, AND NOT SEEING HERSELF REPRESENTED AND HOW THAT LEAVES HER FEELING NOT INCLUDED. AND YOU'VE BEEN IN BUILDING ONE, THIS IS DOWN THE HALL FROM NIH DIRECTOR'S OFFICE, THE ONLY BIT OF DIVERSITY BEING DR. HEALEY, FIRST FEMALE DIRECTOR. THIS IS THE CLINICAL CENTER, WITHOUT MUCH IN THE WAY OF DIVERSITY UNTIL RECENTLY AS YOU CAN SEE THIS IS A NEW INSTALLATION WITH PEOPLE WEARING FACE COVERINGS FOR THE PANDEMIC. THIS PROJECT ATTEMPTS TO ADD TO VISUALS, TAKING ADVANTAGE OF THE GREAT VARIETY OF PEOPLE NECESSARY FOR SUCCESS IN THE BIOMEDICAL RESEARCH ENTERPRISE, SCIENTISTS, NON-SCIENTIST, EARLY CAREER PEOPLE, LATE CAREER INDIVIDUALS, A VARIETY OF TEXTURES AND COLORS TO REPRESENT THAT RICHNESS, THIS IS BUILDING 31, THIS IS JUST OUTSIDE MY OFFICE. I'M ON THE THIRD FLOOR OF BUILDING 1, ABOVE THE NIH DIRECTOR OFFICE. AS YOU TURN TO THE LEFT, THIS LOVELY DISPLAY OF INSTITUTE AND CENTER DIRECTORS, I NEED TO TAKE A MOMENT TO CREDIT FRANCIS COLLINS FOR THIS. WHEN I ARRIVED IN 2008 IT WAS NOT MUCH VARIETY AMONG THE INSTITUTE AND CENTER DIRECTORS. FRANCIS STEPPED DOWN IN DECEMBER. AS OF NOW THERE ARE TEN WOMEN INSTITUTE AND CENTER DIRECTORS, TWO AFRICAN AMERICANS, TWO HISPANIC LATINOS, THREE ASIANS, QUITE A BIT OF VARIETY. THAT'S AT THE VERY TOP. THERE'S A LOT OF WORK TO DO BELOW THAT LEVEL AND BEYOND. BUT ATTESTATION THERE'S ACTION BEHIND THE WORDS. ANOTHER EXAMPLE OF THE ACTION IS WHAT IS BEING EXPECTED OF INSTITUTE AND CENTER DIRECTORS THIS FISCAL YEAR, STARTING OCTOBER 1, THE PERFORMANCE PLAN, THERE'S SOMETHING ABOUT LEADING CHANGE, BUSINESS ACUMEN, SOMETHING ABOUT LEADING PEOPLE. UNDER THE LEADING PEOPLE REQUIREMENT THERE'S NOW -- THERE ARE EXPECTATIONS IN THE DEIA SPACE. HHS PUT FORWARD TWO EXPECTATIONS, NIH DOUBLED THAT. AND AMONG THEM IS THE REQUIREMENT OF RACIAL AND ETHNIC EQUITY PLAN THAT EVERY INSTITUTE AND CENTER WAS TO DEVELOP THIS FISCAL YEAR. NOT ONLY AN I.C. DIRECTOR'S PLAN, IT'S IN EVERY STAFF MEMBER'S PLAN, CASCADES DOWN, SO THEY CAN DO THINGS TO HELP I.C. DIRECTOR BE SUCCESSFUL. SO, IN TERMS OF THESE RACIAL AND ETHNIC EQUITY PLANS, THE EXPECTATION IS I.C.s HAVE DONE A CUSTOMIZED PLAN, THEY STEP BACK, LOOK AT THEIR ENVIRONMENT, WHERE THERE MAY BE INEQUITIES AND DEVELOP PLANS, LONG-TERM AND SHORT-TERM WITH MEASURES OF THINGS THEY WERE GOING TO DO TO MAKE A DIFFERENCE. THAT THEY WOULD IMPLEMENT THOSE THINGS, LOOK AT THE OUTCOMES, ANALYZE PROGRESS, LEARN FROM THAT, AND LOOK AT REVISING THAT ON AN ANNUAL BASIS. THIS IS REQUIRED INVESTMENT OF TIME, INTELLECTUAL CAPITAL, MONEY. GUIDANCE WAS GIVEN NOVEMBER 4. BETWEEN NOVEMBER 4 AND APRIL 1 ICs WORKED HARD, ALL SUBMITTED BY THE APRIL 1 DEADLINE. VERY THOROUGH. PEOPLE LEANED IN. MY OFFICE ALONG WITH OFFICE OF EQUITY, DIVERSITY, AND INCLUSION AND HUMAN RESOURCES WAS TASKED WITH DOING FIRST LEVEL OF REVIEW GIVING FEEDBACK TO THE ACTING PRINCIPAL DEPUTY DIRECTOR ABOUT THESE. WE WERE WERE OVERWHELMED. EVERYONE WAS AT LEAST 15 PAGES, ON AVERAGE 30 TO 45 PAGES, THERE ARE 28 OF THEM, IT TOOK US A WHILE BUT WE'VE BEEN ABLE TO GET GIVE PRELIMINARY FEEDBACK TO PRINCIPAL DEPUTY DIRECTOR. WE'RE NOT GOING TO MAKE THIS DEADLINE, IT'S ALREADY ALMOST THE END OF MAY. HOPEFULLY EARLY JUNE, THIS WILL GET FORMAL FEEDBACK ABOUT THEIR REAPs, INFORMALLY EVERYONE HAS DONE A NICE JOB. THERE'S SOMETHING CALLED OFFICE OF PERSONAL MANAGEMENT MATURITY MODEL AGAINST WHICH THEY WERE MEASURED. LEVEL 1 YOU CHECK THE BOX. LEVEL 2, PRETTY GOOD. LEVEL 3 VERY WELL DEVELOPED. NO ONE JUST MADE A LEVEL 1. AND WHAT'S HAPPENING NOW IS OFFICE OF GENERAL COUNSEL WAS REVIEWING TO MAKE SURE WE DIDN'T OVERSTEP LEGAL BOUNDARIES, SO THAT WHEN THE I.C. DIRECTORS GET FEEDBACK THEY WILL GET A READOUT OF WHERE THEY STAND ON THIS OPM MODEL. THEIR OWN DATA, AND THEN DATA FOR THE OTHER I.C.s ANONYMIZED, PRETTY COMPETITIVE GROUP OF I.C. DIRECTORS WILL BE INTERESTING TO SEE HOW THAT PLAYS OUT. THEY WILL GET A PRESENTATION LATE JUNE OF COMMON THEMES AND BEST PRACTICES, AND I WILL BE MEETING WITH THEM INDIVIDUALLY TO SEE WHAT THE BARRIERS AND OPPORTUNITIES ARE THAT THEY HAVE SEEN HERE. I.C.s ARE NONETHELESS GETTING STARTED IN MANY BECAUSE BY THE END OF THIS FISCAL YEAR THEY ARE SUPPOSED TO STEP BACK, SEE WHAT THEY HAVE BEEN ABLE TO ACCOMPLISH IN THE FEW MONTHS, PROJECT FOR THE COMING FISCAL YEAR. A REALLY EXCITING OPPORTUNITY HERE TO MAKE FOR LASTING INSTITUTIONAL CULTURAL CHANGE. TO MAKE FOR SETTING EQUITY FOR ALL. AND THEN THE EXTERNAL WORKFORCE, WHAT MOST -- OF GREATEST INTEREST TO YOU, THOSE THINGS WE'RE DOING INTERNALLY CAN BE ADOPTED IN OTHER SETTINGS. THERE'S THE FACULTY INSTITUTIONAL RECRUITMENT FOR SUSTAINABLE TRANSFORMATION INITIATIVE, TO CREATE CULTURES OF INCLUSIVE EXCELLENCE, SOMETHING YOU HEAR OVER AND OVER AGAIN BUT LITERATURE SHOWS THIS MAKES A SIGNIFICANT DIFFERENCE IN THE ABILITY TO RECRUIT AND RETAIN PEOPLE FROM MULTIPLE BACKGROUNDS. THIS IS ALSO MODELED AFTER THE DISTINGUISHED SCHOLARS PROGRAM. WHAT'S HERE IS OPPORTUNITY FOR ACADEMIC RESEARCH INSTITUTIONS TO DO FACULTY COHORT HIRING. AGAIN, THE DATA SHOWS THAT BRINGING IN GROUPS OF SCIENTISTS AS OPPOSED TO ONE-OFF RECRUITMENTS TENDS TO BRING IN GREATER DIVERSITY, AND TENDS TO HAVE REINFORCING COMMUNITY THAT'S SO IMPORTANT FOR RETAINING PEOPLE. IT ALSO CALLS FOR INSTITUTIONS TO DO THEIR OWN ASSESSMENT OF WHETHER THERE'S BIAS, EQUITY, WORK LIFE ISSUES, AND THE YEAR 21 SOLICITATION THERE WAS OPPORTUNITY FOR COORDINATION AND EVALUATION CENTER. THIS IS A COMMON FUND INITIATIVE, WITH COMMITMENT OF UP TO $241 MILLION OVER THE NEXT NINE YEARS. SO, WITH THE FIRST, NO PUN INTENDED, FIRST GROUP OF COHORTS WAS FUNDED IN FISCAL YEAR 21 DOLLARS, HIGH AND LOW RESOURCE INSTITUTIONS, COLLABORATION BETWEEN BETWEEN TUSKEGEE UNIVERSITY AND UNIVERSITY OF ALABAMA BIRMINGHAM. STAFF ARE LOOKING AT SUMMARY STATEMENTS, WE'LL GET A LIST IN THE NEAR FUTURE. I THINK THE COMPETITION IS CLOSED OR MAY BE CLOSED TO CLOSING. AT THE END OF THIS WE'LL HAVE AT LEAST 14 COHORTS, APPROXIMATELY 140 EARLY CAREER SCIENTISTS WHO ARE GOING TO BE SUPPORTED AT THESE VARIOUS INSTITUTIONS, BECOME SUCCESSFUL SCIENTISTS, TO COMPETE SUCCESSFULLY FOR NIH FUNDS. MORE MOREHOUSE WILL TRACK THIS AND HELP US FIGURE OUT WHAT ARE GENERALIZABLE PRINCIPLES FOR THIS THAT COULD BE PUT INTO OTHER FUNDING OPPORTUNITY ANNOUNCEMENTS. I ALLUDED TO THE PLAN TO ENHANCE DIVERSE PERSPECTIVES. THIS IS SOMETHING THAT WAS BROUGHT FORWARD IN MARCH OF 21 FROM THE NIH-WIDE BRAIN INITIATIVE. IT LED TO A LOT OF EXCITEMENT FOR THOSE INVOLVED WITH NIH AND BEYOND BECAUSE FOR THE FIRST TIME IT GIVES PEER REVIEWERS THE OPPORTUNITY TO CONSIDER DIVERSITY OF SCIENTIFIC TEAM AS WELL AS SCIENCE WHEN REVIEWING BECAUSE IT'S PART OF THE SCORING. DIVERSITY IS DEFINED BROADLY, DIVERSITY OF DISCIPLINE, GEOGRAPHY, KEEPING WITH NOTICE OF SPECIAL INTEREST. AND IT'S BEING RECOMMENDED THAT THIS BE IMPLEMENTED FOR MULTIPLE OTHER FUNDING OPPORTUNITY ANNOUNCEMENTS. NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCE IS AGGRESSIVE AS ARE OTHERS. WE HAVE PLANS FOR EVALUATION, AS THE DIRECTOR OF THE CENTER FOR SCIENTIFIC REVIEW POINTS OUT WE NEED TO SEE WHETHER HUMAN BEHAVIOR OR HOW HUMANS WILL BEHAVE WITH THIS CRITERION, SHE ASKS THE QUESTION WILL A NOBEL LAUREATE WHO DOESN'T DO AS WELL WITH THE PEDP BUT OTHERWISE HAS EQUIVALENT, A NON-NOBEL LAUREATE, WHO WILL GET THE BETTER SCORE? ALSO COMING FROM UNITE. OTHER THINGS ONGOING, SOMETIMES HARD TO KEEP UP EVEN THOUGH I'M A CO-LEADER OF THIS INITIATIVE. THERE'S SOMETHING CALLED THE SCIENCE EDUCATION PARTNERSHIP AWARD PROGRAM, THAT HAS WILL BE LED BY NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES, IT'S AN OPPORTUNITY TO DO OUTREACH TO K-12 STUDENTS TO GIVE THEM MORE EXPOSURE TO STEM. WE KNOW K-12 POPULATION IS DIVERSE, AS COMPARED TO POPULATION GETTING R01 GRANTS IN NIH. SCIENCE IDENTITY IS OFTEN IN PLACE BY MIDDLE SCHOOL. SO THE VALUE OF DOING MORE OF THIS WAS SUGGESTED. AND WE NOW HAVE ABOUT 17 INSTITUTES, CENTERS, AND OFFICES WHO SIGNED ON TO THIS INITIATIVE. WE ALSO HAVE FOAs AND PRIZEs UNDER DEVELOPMENT. THERE'S AN FOA FOR CLIMATE SELF-STUDIES, INITIAL FUNDS TO HELP ADDRESS THINGS IDENTIFIED UNDER DEVELOPMENT. I LIKE TO THINK OF IT AS FIRST LIGHT. THERE IS UNDERDEVELOPMENT OF STRUCTURED INSTITUTIONAL NEEDS ASSESSMENT, ACTION PLAN, DEVELOPMENT GRANTS FOR MINORITY-SERVING INSTITUTIONS. THERE IS UNDER DEVELOPMENT INSTRUMENTATION GRANT FOR MINORITY-SERVING INSTITUTIONS. EXPANSION OF ADMINISTRATIVE SERVICES AND ACTIVITIES PROGRAM IN BUILD FOR MINORITY-SERVING INSTITUTIONS, EXCELLENCE IN D.E.I. INVESTIGATOR AWARD UNDER DEVELOPMENT. THIS IS DIFFERENT FROM THE ADMINISTRATIVE SUPPLEMENT MY OFFICE HAS OFFERED BECAUSE THIS IS GOING TO BE A DE NOVO COMPETITIVE GRANT THAT PEOPLE CAN APPLY FOR WHETHER YOU'VE HAD NIH FUNDING OR NOT. THE ADMINISTRATIVE SUPPLEMENT YOU HAVE TO ALREADY HAVE NIH FUNDING. BUT ALL OF THESE WERE PRESENTED AT DECEMBER 10 ADVISORY COMMITTEE TO THE DIRECTOR MEETING. IN CONCEPT. AND THEY ARE ALL UNDER DEVELOPMENT. ANOTHER THING THAT WAS PRESENTED, THAT IS WELL UNDERWAY, IS A PRIZE COMPETITION FOR INSTITUTIONAL EXCELLENCE AND DIVERSITY, EQUITY, AND INCLUSION AND ACCESSIBILITY. AGAIN, DEIA. MY TEAM IS TAKING THE LEAD ON THIS ON BEHALF OF THE UNITE E COMMITTEE AND ALL OF NIH. WE HAVE MORE THAN HALF THE INSTITUTES AND CENTERS WHO SIGNED ON TO BE PARTNERS IN THIS. THE INTENT HERE IS TO ACKNOWLEDGE TRANSFORMATIVE CULTURES AND PROCESSES THAT HAVE BEEN DEVELOPED TO ACHIEVE INCLUSIVE EXCELLENCE. AND TO HIGHLIGHT PRACTICES THAT HAVE RESULTED IN MEASURABLE CHANGE AND CREATED MORE INCLUSIVE ENVIRONMENT FOR STUDENTS AND FACULTY. WE CURRENTLY HAVE AN RFI THAT'S OUT. AND REALLY SEEK YOUR INPUT RELATIVE TO THE STRUCTURE OF THE COMPETITION. HOW WE SHOULD GO ABOUT OUTREACH, JUDGING, TIMING, HOW WE SHOULD DISSEMINATE WINNING SUBMISSIONS AND WHAT ARE POTENTIAL BARRIERS FOR INDIVIDUALS OR ORGANIZATIONS TO APPLY. DEADLINE IS JULY 28. AND PRESUME WE GO CAN QUICKLY PROCESS THAT INPUT, WE'RE LOOKING TO POTENTIALLY ANNOUNCE PRIZE COMPETITION FOR FISCAL YEAR 23. AS I SAID, THIS IS 50,000-FOOT LEVEL, THERE'S A LOT MORE GOING ON. I WOULD ENCOURAGE YOU TO TAKE A LOOK AGAIN URL IS NIH.GOV, ENDING STRUCTURAL RACISM, OR GOOGLE NIH UNITE. WATCH THE MEETING ON FRIDAY JUNE 10, WHAT'S PROJECTED FOR THE NEXT SIX MONTHS. I WILL CLOSE WITH OUR FAVORITE ADAGE. GREAT MINDS THINK DIFFERENTLY. THANK YOU. >> THANK YOU SO MUCH, DR. BERNARD. THAT WAS A GREAT PRESENTATION, FULL OF A LOT OF INFORMATION. I WANTED TO ADD A FEW THINGS THAT ERIC AND PENNY AND TEAM, AND WE ALSO CORRECTED TO THE FIRST INITIATIVE. WE'RE ALSO GOING TO SUPPORT IDEAS AROUND THIS AS WELL. A LOT OF THINGS WE'RE LEARNING FROM WHAT DR. BERNARD AND THE UNITE TEAM IS DOING, I THINK IT'S BEEN REALLY A WONDERFUL INTERCHANGE ACROSS THE I.C.s OF SEEING WHAT OTHERS ARE DOING, WHAT WE CAN DO TOGETHER AND WHAT WE CAN ADOPT AS WELL. THANK YOU FOR THAT PRESENTATION. I WOULD LIKE TO TURN IT OVER TO THE COUNCIL FOR ANY COMMENTS OR DISCUSSION. ANY QUESTIONS, PLEASE USE YOUR RAISE HAND FUNCTION OR JUST GO AHEAD AND ENTER IT INTO THE COMMENTS SECTION. PAUL HARRIS, YES, PLEASE. >> DR. BERNARD, THANK YOU SO MUCH. THAT WAS REALLY AN INTERESTING OVERVIEW. MY QUESTION IS WHAT HAS SURPRISED YOU THE MOST IN YOUR WORK OVER THE LAST YEAR AND A HALF? AND MY SECOND QUESTION, WHAT KEEPS YOU UP AT NIGHT? >> OH, WONDERFUL. SO, THE THING THAT HAS SURPRISED ME HAS BEEN HOW MUCH MOMENTUM THERE IS. WE HAD THE SERIES OF DIRECTOR MEETINGS IN THE SUMMER OF 2020 THAT LED TO INTERNALLY UNVEILING UNITE AND LATER EXTERNALLY UNVEILING IT. YOU HAD THAT ALIGNMENT FROM THE UPPER LEADERSHIP. BUT YOU HAVE SO MANY STAFF MEMBERS WHO ARE FULLY ENGAGED IN THE LINE. UNITE ITSELF HAS ABOUT 85 VOLUNTEERS INVOLVED WITH IT. WE HAVE THE SUBCOMMITTEE FOR UNITE, CALLING THE ANTI-RACISM STEERING COMMITTEE, MORE THAN 500 MEMBERS. PEOPLE ARE EXCITED. THEY FEEL THIS IS A SPECIAL TIME, THAT CHANGE CAN BE MADE. THEN WE HAVE EXECUTIVE OFFERED. THE BIDEN ADMINISTRATION PUT THROUGH A LOT OF THESE EXECUTIVE ORDERS TO HAVE US FOCUS IN THIS DIRECTION. EVERYTHING IS ALIGNED. AND I THINK WE'RE -- I HEAR FROM PEOPLE WHO HAVE BEEN AT NIH MUCH LONGER THAN ME, THAT THIS IS JUST DIFFERENT. IT'S NEVER BEEN AS FOCUSED. WHAT KEEPS ME UP AT NIGHT? IT'S A WONDERFUL MOMENTUM. CAN WE KEEP IT UP? WILL THINGS HAPPEN IN THE FUTURE, SINCE WE'RE PART OF THE EXECUTIVE BRANCH THAT MIGHT SLOW THINGS DOWN? BUT MY VIEW OF IT IS WE TAKE ADVANTAGE OF OPPORTUNITIES AS THEY ARE. AND THE REASON THAT I THINK ANY -- MOST OF WHAT WE'RE DOING IS GOING TO BE REALLY ENDURING IS WE'RE LOOKING IN AN EVIDENCE-BASED FASHION, LOOKING ACROSS LANDSCAPE, MAKING IT AN EQUITABLE AND WELCOMING ENVIRONMENT FOR ALL. WE KNOW THAT THIS IS GOING TO MAKE DIFFERENCE IN THE QUALITY OF THE SCIENCE. SO, EVEN OUR WORRY, MY WORRY IS ALLEVIATED BY BEING AWARE OF THOSE THINGS. >> MATIAS. >> IT'S INSPIRING TO SEE THE ACTIVITIES. THEY ARE NEEDED, AS OUR SOCIETY IS SO POLARIZED AND IN NEED OF HEALING. WE AS ONE OF THE FEW REMAINING INSTITUTIONS HAVE TO PLAY THAT ROLE GOING FORWARD. AS YOU CAN HEAR FROM MY ACCENT, I'M A RECENT IMMIGRANT. THAT'S ANOTHER REASON I FEEL STRONGLY. I'M HAPPY TO BE INCLUDED IN THE OVERALL INCLUSION EFFORT. YES, WE'RE VERY UNIQUE MODEL, WHAT HUMANKIND CAN BE ON THIS PLANET. AND BUILDING FORWARD, RECOGNIZING THE URGENT NEEDS WE'VE FALLEN SHORT OF THESE PROMISES PARTICULARLY TO PEOPLE INSIDE THIS COUNTRY BUT ALSO GLOBALLY I THINK IS REQUIRED. I SHARE YOUR CONCERN SIGNIFICANTLY AND IF WE MANAGE TO INSULATE THIS ACTIVITY FROM THE ENVIRONMENT, I THINK WHEREVER POSSIBLE, THE IMPACT CAN BE SUSTAINED AND LASTING WHICH IT NEEDS TO BE. IT'S EXCITING TO SEE THAT ON SO MANY FACETS, MOVING FORWARD. AND WE CAN HELP AND FACILITATE EFFORTS AND OUR AMERICAN SOCIETY OF NEPHROLOGY RELEASED TWO DAYS AGO ACTUALLY A PLEA TO ALL OF US IN THIS COMMUNITY WHICH HAS A BROAD OUTREACH INTO REALLY A LOT OF UNDERSERVED POPULATIONS, IRRESPECTIVE OF RACE, TO TAKE THE EFFORT FROM FEDERAL, REGION, LOCAL LEVELS, I WAS HAPPY TO SEE HAVE YOU VERY STRONG STEM EFFORTS IN PLACE HERE AS WELL BECAUSE THAT'S I THINK URGENTLY NEEDED. THANKS SO MUCH. >> THANK YOU. ANY OTHER COMMENTS OR QUESTIONS? FOR MARIE? >> I WILL SAY TOO THERE WERE A FEW COMMENTS IN THE CHAT BOX, MARIE, THANKING YOU FOR THE OVERVIEW AND ACROSS DEIA. >> IT TAKES A TEAM, WE HAVE A BIG TEAM, THANK YOU. >> THANKS, BECKY. >> A FOLLOW-UP COMMENT. I CAN'T TURN MY CAMERA ON BECAUSE IT'S BROKEN SO I'LL BE INVISIBLE BUT ABLE TO TALK. YOU KNOW, THIS IS ONE OF MANY TIMES I'VE HEARD YOU SPEAK, AND EACH TIME I GET EXCITED. I THINK THE MOST IMPRESSIVE PART OF MANY OF THESE PROGRAMS IS REALLY PUTTING THAT SCIENTIFIC PERSPECTIVE OF ACTUALLY STUDYING WHAT'S EFFECTIVE. AND SOMETIMES WE HAVE MANY GOOD INTENTIONS BUT WE ACTUALLY DON'T KNOW IF THOSE ARE MAKING A SUBSTANTIAL DIFFERENCE, MOST EFFECTIVE AND EFFICIENT, HAVE THE HIGHEST QUALITY, ENGAGE THE BROADEST COMMUNITY. I COMMEND YOUR GROUP AND WOULD ENCOURAGE YOU TO CONTINUE VERY MUCH IN THAT NCATS APPROACH TO EMBED THAT SCIENTIFIC TRANSLATIONAL SCIENCE METHOD. BUT HERE TAKING IT AROUND THAT, THAT BROADER AREA OF DEIA, BECAUSE I THINK THAT THAT WILL REALLY FACILITATE US UNDERSTANDING IN WHAT SITUATIONS, WHAT'S THE BEST WAY TO APPROACH, HOW DO WE REALLY DO THIS IN A -- YOU KNOW, TAKING THE PERSONALIZED HEALTH APPROACH, HOW DO WE ALSO DO THIS TO MAKE SURE OUR INTENT IS AS BROAD AS POSSIBLE, IN FACT THAT ALL OUR VOICES, WHETHER IT'S DATA EQUITY OR WHETHER IT'S INCLUSION, OR WHETHER IT'S THE WAY WE DESIGN OUR STUDIES, ALL THE WAY TO HOW DO WE DISSEMINATE AND COMMUNICATE, AND HELP THAT DISSEMINATION AND IMPLEMENTATION SCIENCE MOVE FORWARD, IS JUST SO CRITICAL. AND, YOU KNOW, I LOOK FORWARD TO -- I'M SURE EVERYBODY HERE DOES -- WORKING TOGETHER BECAUSE WE ALL HAVE A REAL COMMITMENT TO THIS MOVING FORWARD. >> THANK YOU. >> THANKS FOR THOSE COMMENTS, BECKY. I COULDN'T AGREE MORE. YOU KNOW, I DO THINK THAT A LOT OF THESE EFFORTS THAT NIH IS DOING, THAT MARIE TALKED ABOUT TODAY, AND THERE'S SO MANY MORE. CAN'T COVER EVERYTHING. BUT TO ALSO SEE THEM START TO REALLY PLAY OUT IN THE COMMUNITIES THAT WE'VE BEEN INTERACTING WITH FOR SO LONG, SEEING THESE EFFORTS START TO REALLY SEE THAT DIFFERENCE IN CHANGE HAPPENING. AND I THINK THAT THAT'S SUCCESS, IF YOU WILL, THAT WILL BEGET SUCCESS, WILL BEGET MORE OF THESE KINDS OF ACTIVITIES. OF COURSE, THAT'S WHAT WE'RE STRIVING FOR. THANK YOU FOR THOSE COMMENTS. ANY OTHER COMMENTS AT THIS POINT? OKAY. WELL, IT LOOKS LIKE THERE ARE NO FURTHER COMMENTS FOR YOU. THANK YOU SO MUCH FOR THIS PRESENTATION, MARIE. I WOULD IMAGINE THAT WE WILL BE ASKING YOU BACK IN THE FUTURE AS WE CONTINUE TO WORK THROUGH A LOT OF THESE ACTIVITIES, AND GET SOME UPDATES ALONG THE WAY. SO WE LOOK FORWARD TO THAT TIME. UNTIL THEN, THANK YOU AGAIN FOR YOUR PRESENTATION. AND ALL THE WORK THAT YOU'RE DOING. OKAY. I THINK -- NEXT ON THE AGENDA IS OUR PROGRAM UPDATE FROM DPI, LIZ OTTINGER. WE'RE AHEAD OF SCHEDULE. LIZ, I WANT TO CHECK IN. I SEE YOU'RE ON HERE. ARE YOU OKAY WITH MOVING FORWARD OR SHOULD WE TAKE A QUICK BREAK AND THEN RESUME? >> HI, JONI. I'M FINE WITH WHATEVER IS BEST FOR EVERYONE. >> OKAY. WHAT DO PEOPLE THINK, A QUICK FIVE-MINUTE BREAK OR PUSH THROUGH? >> PUSH THROUGH. >> I SEE THUMBS UP, PUSH THROUGH. OKAY. ANNA, ARE YOU GOOD WITH US PUSHING THROUGH? I THINK YOU'RE OVERRULED. >> I WAS CHECKING WITH LIZ TO SEE IF WE COULD GO EARLY SO WE'RE GOOD TO GO. >> I'LL INTRODUCE LYNN OTTINGER, HEAD OF PROJECT MANAGEMENT, DIVISION OF PRE-CLINICAL INNOVATION AT NCATS. WE'RE EXCITED TO HAVE HER HERE TODAY TO GIVE AN UPDATE. LIZ, I'LL TURN IT OVER TO YOU. THANK YOU FOR BEING HERE. >> THANKS. HI, EVERYONE. GOOD AFTERNOON. IF YOU GO TO THE NEXT SLIDE, AT THE LAST COUNCIL MEETING IN JUNE 2021, SCIENTIFIC DIRECTOR OF THE DIVISION OF PRE-CLINICAL INNOVATION, DR. ANTON SENINOF PRESENTED A LARGER OVERVIEW. I WANT TO REORIENT TO DIFFERENT BRANCHES THAT MAKE UP OUR DIVISION, THE STRUCTURE. WHAT I'M GOING TO FOCUS ON IS THE THERAPEUTIC DEVELOPMENT BRANCH YOU SEE IN GREEN. THAT IS LED BY DR. DON LO. I'LL GIVE AN OVERVIEW OF HOW WE OPERATE WITHIN THE BRANCH AND FOCUS ON SPECIFIC EXAMPLES OF HOW WE ADVANCE THERAPEUTIC DEVELOPMENT PROJECTS AND INITIATIVES AND FOCUS ON RARE AND NEGLECTED DISEASE SPACE. I'D LIKE TO SHOW OUR PROCESS, OUR PROGRESS, OUR CONTRIBUTIONS, AND THEN THE VALUE THAT WE'VE CREATED AND TO DO THAT BY ILLUSTRATING SOME EXAMPLES OF OUR PROJECTS. SO IF YOU GO TO THE NEXT SLIDE. THIS IS AN OVERVIEW OF OUR STRUCTURE. AND SO AS I THINK JONI AND OTHERS HAVE POINTED OUT FOR DPI, WE DO A LOT THROUGH COLLABORATION, THAT'S OUR THEME. AND THE DEVELOPMENT OF PLATFORMS. THIS IS OUR PRE-CLINICAL DEVELOPMENT PLATFORM. AND WHAT YOU CAN SEE HERE IS WHAT WE HAVE, WE HAVE DIFFERENT FUNCTIONAL GROUPS THAT WORK TOGETHER ON OUR PROJECTS FROM EARLY DISCOVERY, OUR BIOLOGY, CHEMISTRY GROUPS, WE HAVE THE DMPK, ALL THE DIFFERENT AREAS IN DRUG DEVELOPMENT. WE HAVE THE EXPERTISE IN ORDER TO WORK TOGETHER ON PROJECTS. WE HAVE SAFETY AND TOXICOLOGY GROUP. A CHEMISTRY, INFORMING AND CONTROL GROUP, AND PROJECT MANAGEMENT AND REGULATORY SUPPORT. BELOW YOU SEE THE CHEVRON, TYPICAL WAY A DRUG DEVELOPMENT PATHWAY IS DEMONSTRATED AS SHOWN. I JUST WANT TO FOCUS THAT MOST OF OUR PROJECTS FOCUS IN THE LEAD OPTIMIZATION AND PRE-CLINICAL DEVELOPMENT SPACE, AS YOU CAN SEE HERE, CIRCLED IN RED. AND I JUST WANT TO POINT OUT THAT AS I SAID BEFORE, THESE GROUPS ARE HIGHLY INTEGRATED. IN ORDER TO WORK TOGETHER ON THE PROJECTS, WE TRY TO HELP WITH PROJECTS THAT HAVE HIGH TRANSLATIONAL IMPACT. NEXT SLIDE. THIS IS A CURRENT PICTURE, SNAPSHOT OF OUR THERAPEUTIC DEVELOPMENT PORTFOLIO. ON THE LEFT-HAND SIDE YOU CAN SEE DIFFERENT PROGRAMS IN THE THERAPEUTICS DEVELOPMENT BRANCH. AGAIN, HOW THESE PROGRAMS FUNNEL THROUGH THE DIFFERENT PHASES OF DEVELOPMENT TO OUR ULTIMATE MISSION OF ADVANCING TRANSLATION TO BRING NEW THERAPIES TO PEOPLE. AND IF YOU LOOK BELOW, WHAT YOU CAN SEE IS THE NUMBER OF PROGRAMS THAT WE HAVE IN THE DIFFERENT PHASES. AND, AGAIN, FOCUSING ON PRE-CLINICAL DEVELOPMENT, WE CURRENTLY HAVE ABOUT 15 PROGRAMS, IN THAT SPACE. WE ALSO IF YOU LOOK DOWN BELOW HAVE SEVERAL OTHER INITIATIVES AND BIOLOGICAL DEVELOPMENT PROGRAMS THAT ARE NOT PART -- NOT SEPARATE DRUG DEVELOPMENT PROGRAMS BUT ARE TRANSLATIONAL INITIATIVES TO HELP SUPPORT DRUG DEVELOPMENT. AND I WILL BE TALKING ABOUT ONE OF THOSE AS THE LAST EXAMPLE TO ALSO GIVE YOU AN EXAMPLE THERE. IF YOU GO TO THE NEXT SLIDE, THIS IS JUST TAKING US BACK AGAIN TO THE SPACE THAT WE WORK IN, PRE-CLINICAL SPACE. WHAT I WANTED TO HIGHLIGHT IS THAT IF YOU LOOK, AGAIN, THERE ARE ALL THESE ACTIVITIES THAT TAKE PLACE WITHIN THAT SPACE. AND AS WE ALL KNOW THIS IS NOT A LINEAR SPACE. THERE'S A LOT OF DYNAMIC INTERACTION OF STUDIES AND SCIENCE THAT TAKE PLACE, AND CHALLENGES. A LOT OF SCIENTIFIC AND OPERATIONAL CHALLENGES IN DRUG DEVELOPMENT. AND WHAT I WANTED TO POINT OUT IS THAT AS YOU CAN SEE THERE'S A LOT -- ALL OF THESE ACTIVITIES, WE DON'T NECESSARILY WHEN WE TAKE ON A PROJECT DO ALL OF THESE ACTIVITIES FOR A PARTICULAR PROJECT. BUT IF WE NEED TO, WE CAN TAKE IT -- WE DO TAKE PROJECTS FROM START A LEAD CANDIDATE ALL THE WAY TO THE INVESTIGATIONAL NEW DRUG APPLICATION WHICH IS THE ULTIMATE GOAL TO BE ABLE TO FOLLOW TO TRANSITION OUR PROJECTS INTO CLINICAL DEVELOPMENT. AND THE LAST THING I WANTED TO POINT OUT IS THAT WE DO HELP PROJECTS BECAUSE YOU'LL SEE SOME OF OUR OUTCOMES ARE THAT CLINICAL TRIALS ARE ONGOING. WE DO HELP PROJECTS IN THE CLINICAL TRIAL SPACE, SOME OF THOSE ARE FOR INSTANCE IN THE RARE DISEASE SPACE WE RUN THOSE CLINICAL TRIALS AT THE NIH CLINICAL CENTER, OR WE'LL PROVIDE REGULATORY SUPPORT IF MORE DRUG PRODUCT IS NEEDED OR TOXICOLOGY STUDIES. WE DO PARTICIPATE IN CLINICAL AREAS OF DRUG DEVELOPMENT. IF YOU GO TO THE NEXT SLIDE, I READ A PAPER, TALKING ABOUT WHAT'S THE MAGIC SAUCE OF COLLABORATIONS AND COLLABORATORS, AND FOR US THE MAGIC SAUCE I THINK IS OUR TEAM SCIENCE BASED APPROACH ACROSS NCATS. ALL OF OUR PROJECTS WORK IN THIS FASHION. AND WHAT WE'RE TRYING TO DO IS HAVE A COORDINATED TEAM, AND SO WHAT I'D LIKE TO POINT OUT IS PROJECT MANAGEMENT THERE, BECAUSE THAT'S BEEN A VERY STRONG SUPPORT TO OUR PROJECTS, AND HAVING PROJECT MANAGEMENT SO THAT YOU CAN ACTUALLY KEEP A PROJECT MOVING FORWARD AND DRIVE AND COORDINATE ALL THESE OTHER ACTIVITIES. SO THAT THE PROJECT KEEPS ADVANCING. AND SO, AGAIN, OUR MAIN GOAL IN THIS PRE-CLINICAL PLATFORM IS THAT WE TAKE A PROJECT AND TRY TO PUT THE RIGHT PIECES TOGETHER IN ORDER TO MOVE THAT PROJECT INTO CLINICAL TESTING. SO IF YOU GO TO THE NEXT SLIDE. SO, AGAIN, WHAT I'M GOING TO FOCUS ON IS THE RARE AND NEGLECTED DISEASE PROJECTS IN OUR PORTFOLIO AND GIVE YOU SOME EXAMPLES OF THOSE PROJECTS THAT WE'VE WORKED ON, AND WHAT I WANTED TO POINT OUT WHICH I'M SURE MOST PEOPLE ARE AWARE OF, THERE'S A LOT OF CHALLENGES AND OPPORTUNITIES, AS WE WERE SAYING A LOT OF IT IS SCIENTIFIC AND OPERATIONAL AND REGULATORY CHALLENGES, DRUG DEVELOPMENT, ESPECIALLY IN RARE AND NEGLECTED DISEASE SPACE. WHAT I FOCUSED ON FIRST IS RARE DISEASE PROBLEM. AND IN RARE DISEASES, AS WE ALL KNOW, THERE'S GREATER THAN 7,000 RARE DISEASES, THERE'S ABOUT 30 MILLION PEOPLE IN THE U.S. THAT ARE AFFECTED. THERE'S LESS THAN 10% APPROVED THERAPIES, ABOUT 50% DISEASES THAT CHILDREN ARE AFFECTED. AND SO WITH ALL THESE CHALLENGES, ESPECIALLY WITH THE SMALL RARE DISEASE POPULATIONS, WHICH TRANSLATE INTO CLINICAL TRIAL DIFFICULTIES, ALSO COMMERCIAL DEVELOPMENT DIFFICULTY, AGAIN, AS WE'RE TRYING TO LOOK FOR OPPORTUNITIES, AS THE DEVELOPMENT BRANCH, TO ADVANCE AND HELP RARE DISEASES AND NEGLECTED DISEASE DEVELOP NEW THERAPIES. AS JONI SAYS, YOU KNOW, IT'S MORE THERAPIES TO MORE PEOPLE. AND SO THAT'S OUR MAIN MISSION. IF YOU GO TO THE NEXT SLIDE, THIS IS OUR THERAPEUTICS DEVELOPMENT PORTFOLIO, RARE AND NEGLECTED SPACE. AND WHAT I WANTED TO POINT OUT IS IF YOU LOOK ON THE LEFT YOU CAN SEE THAT GREATER THAN 50% OF THE PROJECTS THAT WE WORK ON ARE EITHER RARE DISEASES WHICH IS ABOUT 49% OF OUR PORTFOLIO, OR NEGLECTED DISEASES WHICH IS ABOUT 5%. AND IF YOU LOOK AT THE PROGRAMS ALSO, THAT'S JUST SHOWING ACROSS THE DIFFERENT PROGRAMS THAT I MENTIONED, YOU CAN SEE THAT WE HAVE, YOU KNOW, RARE AND NEGLECTED DISEASE PROJECTS, MOST OF OUR PROGRAMS. WE ALSO HAVE A WIDE DIVERSITY IF YOU LOOK BELOW IN THERAPEUTIC AREAS, AGAIN WE DON'T FOCUS ON ONE THERAPEUTIC AREA. IT'S REALLY PROJECT DRIVEN, WHERE CAN WE MAKE AN IMPACT, WHAT PROJECT NEEDS HELP IN ORDER TO ADVANCE THE THERAPEUTICS. AND WE ALSO WORK, IF YOU LOOK ON THE RIGHT, IN A CHANGE OF MODALITIES. WE ALSO WORK IN BIOLOGICS, AND WE ALSO WORK AT SMALL MOLECULES. IF YOU GO TO THE NEXT SLIDE AGAIN, THE FIRST EXAMPLE THAT I'D LIKE TO DISCUSS AND HIGHLIGHT IS THE RARE DISEASES DRUG DEVELOPMENT FOR DISEASES OF LOW PREVALENCE WHICH MEANS THERE'S A SMALL NUMBER OF PATIENTS. AND, AGAIN, THE CHALLENGES ARE THE SAME BUT, AGAIN, THIS REALLY HAS TO DO WITH THE SMALL NUMBER OF PATIENTS, THAT'S WHAT I WANT TO FOCUS ON IN THIS FIRST EXAMPLE. THE OPPORTUNITIES OF HOW WE CAN COLLABORATE AND WORK ON A THERAPEUTIC FOR A PROJECT. SO, IF YOU LOOK AT THE NEXT SLIDE, THIS PROJECT IS A TREATMENT FOR DYSPLASIA, LEAD INVESTIGATORS AT MASS GENERAL HOSPITAL. AND THIS IS WHAT YOU COULD SAY IS AN ULTRA RARE DISEASE, LESS THAN 30 KNOWN CASES. IT'S A DISEASE OF BONE DEVELOPMENT AND MINERAL HOMEOSTASIS, INCREASED CALCIUM, IF YOU LOOK ON THE RIGHT WHAT YOU CAN SEE IT'S A RECEPTOR, PARATHYROID HORMONE RECEPTOR TYPE 1, WHICH HAS MUTATIONS WHICH CAUSES THE PROTEIN TO BE IN AN ACTIVE STATE OR TO BE ON. AND THERE'S NO EFFECTIVE TREATMENTS CURRENTLY, AND IF YOU LOOK IN THE UPPER RIGHT WHAT HAPPENS IS THAT THESE CHILDREN, BECAUSE IT AFFECTS CHILDREN AT AN EARLY AGE, THEY HAVE TO UNDERGO MULTIPLE REPEATED ORTHOPEDIC SURGERIES TO CORRECT BONE DEFECTS. AND SO OUR COLLABORATORS AT MASS GENERAL FOUND A POTENTIAL PEPTIDE. IF YOU LOOK AT RECEPTOR, WHAT THIS PEPTIDE DOES, IT ACTUALLY SHIFTS THE RECEPTOR TO THE OFF STATE. AND SO THIS IS A POTENTIAL PEPTIDE DRUG, BUT, AGAIN, THE BIG CHALLENGE IS CURRENTLY THERE'S ONLY ONE ADULT PATIENT THAT'S KNOWN, THAT COULD PARTICIPATE FOR THE PHASE 1/2 TRIAL AROUND NEEDS TO BE TRANSITION TO SMALL NUMBER OF PEDIATRIC PATIENTS. THEY ARE SEVERELY AFFECTED WITH THE BONE DEFECTS. HOW DO WE DO DRUG DEVELOPMENT FOR SUCH A SMALL PATIENT POPULATION? IF YOU GO TO THE NEXT SLIDE. WHAT WE BELIEVE IN HERE AT NCATS AND WHAT OUR WHOLE THEME IS, AGAIN, IS HOW DO WE BUILD A PLAT FORM, COLLABORATIVE ECOSYSTEM. WE'VE BEEN WORKING WITH THE JANSON DISEASE FOUNDATION, THE PATIENT FOUNDATION, WHICH CONNECTS THE KEY PARTNERS, YOU HAVE PATIENTS WHO ENGAGE IN CLINICAL RESEARCH, YOU HAVE THE RESEARCHERS AND CLINICIANS ON THE RIGHT-HAND SIDE THERE, THEY PROVIDE THE SCIENTIFIC AND DISEASE EXPERTISE, CARE FOR THE PATIENTS, AND REALLY THAT LINK BETWEEN THE SCIENCE AND THE PATIENTS. TDB PRE-CLINICAL PROJECT, WE COME IN AND WE'VE BEEN PROVIDING DRUG DEVELOPMENT EXPERTISE TO ENABLE IND COORDINATING ALL PARTS OF THE PROJECT TRYING TO ENABLE THAT TRANSITION FROM PRE-CLINICAL TO CLINICAL. AND THEN WE'RE WORKING WITH THE NIH CLINICAL CENTER, WORKING WITH DR. SHAH AT NIDDK, DR. COLLINS AT NIDCR, TO RUN THE NATURAL HISTORY AND EXECUTE THE CLINICAL TRIALS. AGAIN, IT'S BRINGING ALL THE PIECES TOGETHER FOR THAT PROJECT SO THAT WE CAN MOVE IT AND DRIVE IT FORWARD. IF YOU LOOK AT THE NEXT SLIDE, AGAIN, ANY SORT OF REITERATES WHAT WE SAID BEFORE IN TERMS OF DEVELOPING AN ECOSYSTEM BUT ALSO WHAT WE ALWAYS GO BY, IT'S REALLY IMPORTANT, TO REMEMBER THERAPEUTIC STARTS AND ENDS WITH THE PATIENT, IMPORTANT ESPECIALLY WITH A SMALL NUMBER OF PATIENTS. YOU HAVE TO BE CLOSELY ALIGNED WORKING WITH THOSE PATIENTS IN ORDER TO BE ABLE TO RUN THE CLINICAL TRIALS. AND SO AGAIN THIS PROJECT STARTED WITH OUTREACH, AND WAS DRIVEN BY THE PATIENT FOUNDATION, THERE'S NINA MAZAR, FOUNDER OF JANSEN'S FOUNDATION, CONNECTED RESEARCHERS, CLINICIANS, DRUG DEVELOPERS, REACHED OUT TO TDP AND IS PARTICIPATING IN THE NATURAL HISTORY STUDY AND SO ARE THE OTHER PATIENTS IN THE JANSSEN FOUNDATION. YOU WERE ABLE TO IDENTIFY POTENTIAL THERAPEUTIC CANDIDATE, DID THE INITIAL EFFICACY STUDIES, TESTING IN MOUSE MODEL OF THE DISEASE. YOU SEE ON THE BOTTOM, IF YOU LOOK AT THE GREEN BOX, WHAT YOU CAN SEE IS THE IA IN RED WHEN YOU GIVE THE PEPTIDE TO THE MOUSE MODEL AND ON THE LEFT IS THE VEHICLE. YOU CAN SEE THAT IT CORRECTS SOME OF THE BONE DEFECTS AND MAKES THE BONE LOOK MORE LIKE THE WILD TYPE ON THE FAR LEFT. IN PRE-CLINICAL DEVELOPMENT THE PROGRESS OF THE PROJECT WE'VE COMPLETED THE GMP MANUFACTURING OF THE FORMULATION, FORMULATION OF THE DRUG CANDIDATE, WE'RE CURRENT LIP IN THE GLP TOXICOLOGY, MOVING TOWARD REGULATORY IND SUPPORT. WE'RE ALSO DEVELOPING CLINICAL ASSAYS, AGAIN, TO HELP THAT COORDINATION BETWEEN PRE-CLINICAL AND CLINICAL. SO WE'RE STARTING WITH THE END IN MIND. WE'VE BEEN VERY INVOLVED IN COMMUNICATING WITH THE CLINICIANS AND WITH THE PATIENTS IN ORDER TO DESIGN THE WHOLE PROGRAM ABOUT BEING ABLE TO DO THE PHASE 1 AND PHASE 2, IN THAT N-of-1 PATIENT TO START. AND SO WHAT WE THINK IS CRITICAL THAT WE CAN ALSO CONTRIBUTE HERE AT TBD IS DISSEMINATION TO PROVIDE INFORMATION HOW DO YOU DO -- WHAT IS REGULATORY PATH, HOW DOES THIS OCCUR WHEN WE HAVE THESE SMALL NUMBER OF PATIENTS AND ALSO HOW TO TRANSITION IT TO THE ACTUAL PEDIATRIC POPULATION TO DO A SMOOTH TRANSITION THAT CAN BE TESTED IN THAT POPULATION ALSO, SO WE CAN GET TO THE ULTIMATE GOAL OF HOPEFULLY IMPROVING THE LIVES OF THE PATIENTS AS THIS TREATMENT WORKS. THAT'S ONE EXAMPLE, I'VE TRIED TO SHOW THE ECOSYSTEM THAT WE WORK ON TO DEVELOP OUR PLATFORM AND TO OPERATIONALIZE IT. SO IF YOU LOOK AT THE NEXT SLIDE, I'M GOING TO TALK ABOUT THE NEGLECTED TROPICAL DISEASES BECAUSE WE ALSO WORK IN THIS SPACE. AND THE PROBLEM HERE IS THAT THERE ARE 20 NEGLECTED TROPICAL DISEASES DEFINED BY W.H.O., GREATER THAN ONE BILLION PEOPLE AFFECTED, GREATER THAN TWO BILLION AT RISK. 0.5BILLION CHILDREN AT RISK FROM THESE DISEASE, AFFECTS POOREST AND MOST VULNERABLE PEOPLE. AGAIN, YOU HAVE THOSE SAME CHALLENGES IN THE DRUG DEVELOPMENT SPACE, AND, AGAIN, BECAUSE OF THE LOW-RESOURCE SETTINGS THERE'S ALSO A LOT OF EQUITY, HOW DO YOU GET IT TO ALL THE PATIENTS. AND SO, AGAIN, THE OPPORTUNITIES THAT WE'VE TRIED TO WORK TOWARDS TO ACTUALLY FACE THESE CHALLENGES WE WERE ABLE TO DO A PROJECT IN COLLABORATION, NEXT SLIDE, ABLE TO WORK WITH DNDI, DRUGS FOR NEGLECTED DISEASE INITIATIVE, DEVELOPMENT FOR THE TREATMENT OF SLEEPING SICKNESS. LAURENT FRAISSE IS LEAD INVESTIGATOR, THE FIRST THAT HAS COMMENT OUT OF DNDI. THIS SPREADS, INFECTION THROUGH A FLY BITE, INTO THE BLOODSTREAM, IT TRANSFERS INTO THE CEREBROSPINAL FLUID AND LEADS TO DIFFERENT STAGE OF DISEASE UNTIL YOU HAVE COMA AND DEATH, MOSTLY AFFECTS THE POOREST AND OF IN AFRICA, 8.5 MILLION PEOPLE. THE GOAL OF THE W.H.O. IS THE ACTUAL ELIMINATION OF THIS DISEASE, HOW DO YOU CHANGE THE TREATMENT PARADIGM? AND AGAIN IF YOU LOOK ON THE LEFT HERE CURRENT TREATMENTS HAVE HIGH -- ARE VERY TOXIC, A LOT OF SIDE EFFECTS, AND TREATMENT DURATION AND COST, SO IT'S NOT OPTIMAL TO TREAT ALL PEOPLE. WHAT THEY SLOWLY WORKED TOWARDS IN 2018 IS ORAL TREATMENT, HAVE GOTTEN TO 10-DAY APPROVAL OF FEXINIDAZOLE, A GAME CHANGER, SIMPLER TREATMENT. YOU WANT MORE ACCESSIBILITY AND COMPLIANCE. THE BEST WAY WOULD BE IF YOU HAD AN ORAL SINGLE DOSE DRUG, THAT'S WHAT ACOZIBAROLE WILL PROVIDE. IT'S A DIAGNOSE AND TREAT SO YOU COULD - ONCE YOU KNOW A PERSON HAS IT YOU COULD QUICKLY TREAT IT, AIDING IN ELIMINATION. AND SO OUR INVOLVEMENT IN THIS PROJECT IS, AGAIN, SOMETIMES ONE STUDY, ONE PIECE, ONE PILL MIGHT BE ALL THAT'S NEEDED. AND SO EVEN THOUGH THEY HAD A LOT OF THIS PROJECT ONGOING, IN ORDER TO ACTUALLY REGISTER IT THEY CAME UP AGAINST THAT THEY NEEDED A CHRONIC GLP TOXICOLOGY STUDY AS KEY REQUIREMENT FOR REGULATORY APPROVAL, AND AT THE TIME THEY NEEDED SOME -- THEY WERE ASKING US IF WE COULD PROVIDE THAT RESOURCE. AND SO WE WORKED CLOSELY WITH THEM IN ORDER TO GET THAT STUDY DONE, AND AS A RESULT IN 2021 DNDI INTERNALLY NAMED THIS PROJECT OF THE YEAR IN THE CLINICAL SPACE. THEY ACKNOWLEDGED THEY FELT NCATS TEAM IS GREATLY RESPONSIBLE FOR THIS SUCCESS. AND SO WHERE IS THIS NOW? SANOFI AND W.H.O. ARE PARTNERS TO PROVIDE ACOZIBAROLE FREE THROUGH PUBLIC HEALTH SYSTEMS IN COUNTRIES, DNDI COMPLETED PIVOTAL PHASE 2/PHASE 3 CLINICAL TRIALS, MOVING TOWARDS REGISTRATION. SO, AGAIN, WE FEEL LIKE WE MADE AN IMPORTANT CONTRIBUTION HERE. WE ADDED IMPACT FOR GLOBAL HEALTH. AND IN THIS CASE IT WAS LIKE THIS ONE PIECE THAT WE WERE ABLE TO CONTRIBUTE. SO GO TO THE NEXT SLIDE. NEXT AREA OF, AGAIN, REPRESENTING A PROJECT OF HOW WE CAN AGAIN GET MORE TREATMENTS TO MORE PATIENTS, MORE RAPIDLY, IS REPURPOSING FOR RARE DISEASES. AND SO HERE IT OFFERS AN OPPORTUNITY AND CHALLENGE IF YOU CAN FIND ONE DRUG TO TREAT MULTIPLE DISEASES. I'D LIKE TO GIVE AN EXAMPLE OF A PROJECT THAT WE WORKED ON, NEXT. WE REPURPOSED A EUROPEAN UNIT, THE LEAD IS ALSO A PROFESSOR AT BOSTON UNIVERSITY. , THE GROUP OF INHERITED BLOOD DISORDERS PRIMARILY AFFECT RED BLOOD CELL PRODUCTION AND STRUCTURE, YOU CAN SEE ON THE RIGHT THOSE ARE BLOOD SMEARS. I'M SURE MOST PEOPLE MAY BE FAMILIAR WITH THE SICKLE CELL SHAPE. IT'S A RARE DISEASE, THERE'S ONLY 2,000, SICKLE CELL IS ALSO RARE, THERE'S ABOUT 100,000 CASES IN THE U.S. WHAT YOU CAN SEE, MINORITY POPULATIONS ARE AT HIGH RISK. AND IT'S ALSO A SIGNIFICANT HEALTH PROBLEM IN MORE THAN 160 COUNTRIES WORLDWIDE. GLOBALLY THERE'S OVER 330,000 AFFECTED INFANTS BORN ANNUALLY WITH THESE DISEASES. SO EVEN THOUGH THERE ARE SOME THERAPEUTICS THAT HAVE BEEN IN USE LIKE THERE'S BONE MARROW TRANSPLANT, THERE'S HYDROXYUREA, AND WORKING TOWARDS GENE THERAPY, SOME OF THE CURRENT THERAPIES HAVE A LOT OF SIDE EFFECTS ASSOCIATED WITH THEM AND THEY ARE NOT AS -- AND GENE THERAPY MAY NOT BE AS ACCESSIBLE TO ALL OF THE POPULATION, TO HAVE EQUITY AND CARE. IT'S IMPORTANT TO FIND OTHER MOLECULES. THIS IS A POTENTIAL SMALL MOLECULE. IT'S A DRUG THAT WAS APPROVED GREATER THAN 40 YEARS AGO. IT WAS ONLY PART OF A COMBINATION DRUG SO PART OF A PARKINSON'S -- FOR PARKINSON'S DISEASE APPROVED BY EMA AND HEALTH CANADA. THE QUESTION, AGAIN, IN THIS PROJECT WAS WHAT NEW DATA WOULD BE NEEDED FOR APPROVAL IN HEMOGLOBINOPATHIES? NEXT SLIDE. SO AGAIN HEMOGLOBINOPATHIES, TWO DISEASES, WHAT THEY HAVE IS THEY HAVE MUTATIONS IN THE BETA-GLOBIN GENE OF HEMOGLOBIN, ON THE RIGHT IS MOLECULE WITH HEMOGLOBIN, AND THE BETA SUBUNIT WHERE MUTATIONS OCCUR. THEY HAVE A COMMON TARGET MECHANISM OF ACTION. IF YOU LOOK IN GREEN, THAT IS GAMMAGLOBIN SUBUNIT, IT GOES DOWN AFTER BIRTH, AND BETA-GLOBIN EXPECT GLOWS UP, MUTATIONS TAKE HOLD, ONSET OF SYMPTOMS. IF YOU LOOK ON RED AND FOLLOW THE GREEN, WITH THE WHOLE PREMISE OR THOUGHT TO TARGET THIS COMMON MECHANISM OF ACTION. YOU CAN SEE BY FOUR WEEKS IF YOU GIVE THE BEN, YOU HAVE INCREASE IN FETAL HEMOGLOBIN EXPRESSION. IF YOU GO TO THE NEXT SLIDE, HOW WERE WE, AGAIN, ABLE TO USE OUR PRE-CLINICAL PLATFORM AND ABLE TO REPOSITION THIS TO ACCELERATE DRUG DEVELOPMENT FOR THIS PARTICULAR MOLECULE? IT TURNS OUT THAT A LOT OF THE INFORMATION WAS NOT ABLE TO BE USED BECAUSE, AGAIN, IT WAS 40 YEARS OLD, PART OF A COMBINATION. A LOT OF PRE-CLINICAL STUDIES WE WERE ABLE TO DO, ACQUIRED EXISTING GENE API AND TO MOVE THIS FORWARD AND GET IT TO AN IND, WE STARTED THE PROJECT IN 2014, AND AS YOU CAN SEE BY 2019 WE WERE ABLE TO CLEAR THE INDs, ABLE TO SAFELY PROCEED INTO THE CLINIC AND ALSO IN HEALTH CANADA. AGAIN, WE COLLABORATE WITH OTHER, HOW DO WE BRING THESE PIECES TOGETHER, FOR CLINICAL COLLABORATED, NHLBI PROVIDED THE FUNDING. SO CURRENTLY IT'S CALLED THE BENEFITS TRIAL, PHASE 1B FOR BETA CELL-ASSEMIA, SITES IN THE U.S. AND CANADA. GO TO THE NEXT SLIDE. THERE'S THE NEXT EXAMPLE. THIS IS THE SPACE OF GENE THERAPIES, WITH A LOT OF OPPORTUNITIES FOR RARE DISEASES. 85% OF RARE AND ULTRA RARE DISEASES, PATHOGENIC VARIANTS ARE IN SINGLE GENES THAT REGULATE THAT PRODUCT FUNCTION. AND ON THE LEFT-HAND SIDE THERE AGAIN IN THE PIE WHAT YOU SEE IS 770 DRUGS APPROVED FOR RARE DISEASES, AND THERE'S ONLY 7% SO FAR APPROVED FOR CELL AND GENE THERAPIES. BUT THERE'S A LOT OF OPPORTUNITY AND PROMISE THAT'S BEING HELD THERE IN RARE DISEASES, AND MANY OF THE RARE DISEASES HAVE THAT LOW PREVALENCE AGAIN, WITH THE GENE THERAPY COULD BE A GOOD CURE OR SOLUTION FOR TRYING TO PROVIDE THERAPEUTICS TO THOSE LOW PREVALENCE DISEASES. SO, AGAIN, THERE'S MANY CHALLENGES. AND I JUST WOULD LIKE TO SHOW YOU HOW WE HAVE TRIED TO GO ABOUT PARTICIPATING IN THE DEVELOPMENT OF CELL AND GENE THERAPIES, OVERCOMING SOME CHALLENGES AND PROVIDING OPPORTUNITIES TO THE RARE DISEASE COMMUNITY. SO IF YOU GO TO THE NEXT SLIDE. OUR GOAL IS TO BUILD AN ACCESSIBLE GENE THERAPY PLATFORM. YOU'VE HEARD DR. P.J. BROOKS TALK ABOUT THIS. MANY PROJECTS HE HAS ONGOING IN THE EXTRAMURAL IN 2016 NIH INTRAMURAL ESTABLISHED GENE THERAPY DEVELOPMENT INFRASTRUCTURE ALSO, AND THE THERAPEUTICS FOR RARE AND NEGLECTED DISEASE PARTNERS WITH NCI FREDERICK WHICH HAD MANUFACTURING CAPABILITIES IN ORDER TO WORK ON MANUFACTURING FOR THESE DRUG PRODUCTS. AND SO THERE WERE TWO PROJECTS WE WORKED ON, TRYING TO BUILD UP MANUFACTURING CAPABILITIES, AND TO HAVE LESSONS LEARNED AND UNDERSTAND WHAT'S INVOLVED IN MANUFACTURING GENE THERAPIES. AND SO THERE WAS A GENE THERAPY FOR L-AMINO ACID DECARBOXYLASEDE ENCY, A LEAD COLLABORATOR WITH WHAT IS NOW PPG THERAPEUTICS, WORKING THROUGH THE EEU REGISTRATION OF THIS MOLECULE. IN 2016 WE ALSO WORKED WITH DR. DWIGHT KOEBERL AT DUKE, WAS ASKBIO, TRANSFERRED TO BAYER FOR POMPE DISEASE TO AID IN MANUFACTURING OF THAT DRUG PRODUCT. CLINICAL TRIALS WERE SUPPORTED BY NIAMS. SO, AGAIN, THERE'S A LOT OF COLLABORATION AND COORDINATION AMONG DIFFERENT RESOURCES ACROSS NCATS AND NIH. IN 2017 THERE WAS A CONCEPT CLEARANCE APPROVED BY NCATS COUNCIL WHICH WAS NCATS COLLABORATIVE RARE PLATFORM VECTOR GENE THERAPY TRIAL. AND THIS INVOLVES THREE DIFFERENT DIVISIONS AND OFFICES OF NCATS, WHICH IS THERAPEUTICS DEVELOPMENT BRANCH, OUR NEW DIVISION OF RARE DISEASE RESEARCH AND INNOVATION, WHICH DR. P.J. BROOKS IS NOW THE ACTING DIRECTOR, AND OFFICE OF STRATEGIC ALLIANCE WHICH IS LED BY LILLY FORTILLA. WHAT'S COME OUT OF THAT IS THAT IN 2019 ONE OF THE PROGRAMS THAT'S BEEN INITIATED, I'M GOING TO BE TALKING ABOUT OUR INTRAMURAL CONTRIBUTIONS, THE INITIATION OF THE PAVE-GT PROGRAM. NEXT SLIDE. THIS IS THE PAVE-GT WEBSITE. AGAIN, OUR WHOLE MISSION IS TO TRY TO PROVIDE ACCESSIBILITY. PART OF THAT ACCESSIBILITY IS TO THE INFORMATION SO EVERYBODY CAN DEVELOP A PLATFORM, WHICH IS A ROAD MAP FOR OTHERS SO THEY CAN SEE WHAT HAPPENS DURING THIS DRUG DEVELOPMENT PROCESS AND HAVE ACCESS TO DETAILED INFORMATION. ON THE RIGHT-HAND SIDE WE DID A MARKER PAPER THAT YOU CAN SEE THERE THAT WAS PUBLISHED. AND SO OVERALL WHAT WE'RE TRYING TO DO IS INCREASE EFFICIENCIES, ENHANCE COLLABORATION, LEVERAGE EXISTING RESOURCES AND EXPERTISE, INCREASE ACCESSIBILITY, AS I SAID, AND MOST OF ALL DISSEMINATE BEST PRACTICES AND SHARE INFORMATION. SO THAT THIS CAN ACTUALLY BE A PLATFORM THAT HELPS OTHERS. NEXT SLIDE. SO, AGAIN, HERE'S OUR -- THIS GOAL PERMEATES THROUGHOUT THAT CAN WE USE COMMON PROCESSES EVEN IN THE PRE-CLINICAL SPACE ACROSS THE PLATFORM, AND THEN CAN WE MAKE THAT DATA PUBLICLY AVAILABLE TO MAKE THIS MORE OF A PLATFORM. AND SO, AGAIN, WE'RE COLLABORATING WITH INTRAMURAL LABS, WE'RE COLLABORATING WITH DR. VAN DIDDY AT NHGRI FOR PCMI AND MMAB ON THE LEFT, AND WITH NINDS FOR THE CONGENITAL MYASTHENIC SYNDROMES, WORKING TOGETHER, WE FORMED THIS LARGE TEAM AND THERAPEUTIC DEVELOPMENT BRANCH IS DOING THE FULL IND-ENABLING STUDIES FROM SCALEUP PROCESS AND DEVELOPMENT, GMP MANUFACTURING, TRYING TO DEVELOP PROCESSES, GET STANDARDIZED ASSAYS, AND TO SEE, AGAIN, IF WE CAN THEN TRANSMIT OR SHARE THAT INFORMATION WITH THE COMMUNITY, WE'RE DOING ALL THE DMPK, IND-ENABLING STUDIES ARE ONGOING AND WE'RE WORKING ON THE FIRST PCCA AND OTHER THREE ALSO. AND I JUST WANT TO POINT OUT THESE ARE HUGE TEAMS, IT DOES TAKE A HUGE TEAM TO COORDINATE AND BRING ALL OF THIS TOGETHER, AND THERE'S ABOUT 60 PRE-CLINICAL DEVELOPMENT SCIENTISTS, NCATS, THERAPEUTICS DEVELOPMENT BRANCH WITH THE CROs AND CMOs THAT ARE DOING SOME OF THESE LATE-STAGE STUDIES AND IN COLLABORATION WITH ALL OF THE OTHER INSTITUTES AT NIH THAT ARE WORKING ON THIS PLATFORM TOGETHER. SO IF YOU GO TO THE NEXT SLIDE. AS I SAID, ONE OF OUR KEY DRIVERS HERE IS DISSEMINATION OF OUR ACTIVITIES. WE STARTED WITH THE PLATFORM LAUNCH WHICH YOU'VE SEEN BEFORE, I THINK YOU HEARD FROM THE DIVISION OF RARE DISEASES INNOVATION, RARE DISEASE -- SORRY, I'M LEARNING THE ACRONYM MYSELF. IT USED TO BE ORDR, WEBSITE AND MARKER PAPER. WE'RE TRYING TO DO THIS PRE-CLINICAL PLAYBOOK WHERE WE ALSO WOULD LIKE TO WRITE WHITE COVERS ELABORATING ON KNOW-HOW OF DRUG DEVELOPMENT, SHARING PROGRAM METHODS, PRE-CLINICAL DEVELOPMENT, RELATED SCIENTIFIC ARTICLES SO YOU CAN HAVE A PRE-CLINICAL PLAY BOOK AND REGULATORY SUBMISSIONS AND COMMUNICATIONS WHICH WE THINK ARE ONE OF THE KEY AREAS THAT WE CAN DISSEMINATE. WE ALREADY ESTABLISHED WE GOT ORPHAN ORPHAN DRUG DESIGNATION, WE'RE NAVIGATING, AND CAN SHARE THE COMPLETE APPLICATION THAT WE SUBMITTED. WE HAD AN INTERACT MEETING, WITH THE FDA, THAT'S A PRE-IND MEETING WHERE YOU CAN TALK VERY EARLY ON ABOUT THE DEVELOPMENT PROCESS. SO WE HOPE TO ALSO HAVE A WHITE PAPER AND TO SHARE THAT INFORMATION AND HOW WE WENT ABOUT THAT, WHAT WE SUBMITTED, WHAT WERE THE FDA RESPONSES TO OUR QUESTIONS. AND THEN WE PLAN TO SHARE ALL THE OTHER REGULATORY PACKAGES AND ALL THE INFORMATION AS THESE PROJECTS MOVE FORWARD IN DEVELOPMENT. AND AS THEY MOVE INTO THE CLINIC WE HOPE TO HAVE A CLINICAL PLAYBOOK WHERE WE CAN SHARE BIOMARKERS AND NATURAL HISTORY DOCUMENTATION AND SHARE THE CLINICAL DATA. AND SO, AGAIN, IT'S A PLATFORM TO TRY TO OPERATIONALIZE, TO SHARE THE INFORMATION, TO DISSEMINATE AND HELP THE COMMUNITY. SO, IF YOU GO TO THE LAST EXAMPLE, IN THIS LAST EXAMPLE I WANTED TO TALK, AGAIN, YOU KNOW, OUR MISSION IS ALSO TRANSLATIONAL INITIATIVES AND PROCESS OF TRANSLATION. AND SO I WANTED TO TALK ABOUT THIS GAP BETWEEN THE SCIENTIFIC RESEARCH AND TRANSLATION TO THERAPIES FOR RARE DISEASES. ONE INITIATIVE IN WHICH WE'RE TRYING TO ADDRESS THIS, SO, AGAIN, IF YOU LOOK HERE ON THE LEFT, WHAT YOU CAN SEE FROM A PAPER IN 2018 PUBLICATIONS, SCIENTIFIC INFORMATION IS MUCH GREATER THAN TRANSLATING TO THE ACTUAL THERAPEUTICS AND APPROVALS, IF YOU SEE FOR FDA AND EMA. AND SO THERE'S THIS GAP BETWEEN THE SCIENCE AND ACTUAL APPROVALS FOR RARE DISEASES. AND SO, AGAIN, IT'S THE CHALLENGES AND, YOU KNOW, THE WAY THAT WE'RE THINKING ABOUT THIS THAT DATA EXISTS BUT IT'S NOT COLLECTIVELY ORGANIZED, ALL WE SHARE TO EXTRACT THAT INFORMATION. AND AND SO THE OPPORTUNITIES CAN WE APPLY ADVANCES IN DATA AND INFORMATION TECHNOLOGIES, AND BUILD INFORMATICS PLATFORM TO MAKE THIS RARE DISEASE INFORMATION MORE ACCESSIBLE. AS YOU KNOW, WE HAVE SEVERAL OF THESE ACTIVITIES ACROSS NCATS, AND WHAT WE'RE AGAIN HOPING TO DO IS THAT ALL OF THIS, THEY ARE ALL GOOD INITIATIVES THAT CAN INTEGRATE MORE INFORMATION TOGETHER. FOR INSTANCE, THE GUARD PLATFORM WHICH YOU'VE ALL HEARD ABOUT, IT'S VERY ESTABLISHED, REALLY PROVIDES THE PATIENTS THE INFORMATION THAT THEY NEED WHEN THEY ARE FIRST DIAGNOSED, AND WHAT WE'VE BUILT HERE WHICH I'LL TALK ABOUT IS THAT WE'VE ACTUALLY TRIED TO INTEGRATE SOME OF THE PARTS OF GUARD. OUR ONTOLOGY COMES FROM GUARD. WE'VE BEEN TRYING TO BUILD A RARE SOURCE USING BIG DATA APPROACH TO UNDERSTAND HOW RARE DISEASES ARE GROUP, IF WE CAN GROUP THEM ACCORDING TO THEIR, YOU KNOW, PATHWAYS, CAN WE FIND WAYS OR NEW WAYS THEY CAN BE TREATED BY SIMILAR MOLECULES OR THE SAME MOLECULE. AND SO, AGAIN, WE'RE TRYING TO BRING TOGETHER CURRENTLY THE GENES, VARIANTS, PROTEINS, DISEASE INFORMATION, AND EVENTUALLY WE'LL ADD OTHER INFORMATION BUT, AGAIN, THIS WAS STARTING ON THE MOLECULAR INTERACTION LEVEL, AND SO WE HOPE THAT IT MEETS WITH ALL THE OTHER INFORMATION RESOURCES. AND THAT OTHERS ALSO CAN INTEGRATE THEIR INFORMATION SO THAT WE MAKE THIS VERY INTEROPEARABLE. WE'RE TRYING TO INTEGRATE DATA WE HAVE ACCESS TO. IF YOU GO TO THE NEXT SLIDE, HOW DID WE GO ABOUT THIS? AGAIN, WE'RE TRYING TO CREATE A SEARCHABLE, ACCESSIBLE, USER FRIENDLY RESOURCE. AND WE WERE STARTING IT BY TRAINING THE PLATFORM THROUGH MANUAL CREATION OF SOME OF THE DISEASE LITERATURE. IT WAS BASED ON PROJECTS THAT WE WORKED ON, IN OUR THERAPEUTIC DEVELOPMENT PLATFORM. AND WE HAVE WORKED ON INTEGRATING THE KNOWN PUBLIC DATABASES, AND THEN DEVELOPING MODULES FOR AUTOMATING MINING OF LITERATURE AND EXPANDING THAT TO THE 7,000 RARE DISEASES, AGAIN USING ALL THE -- HAVE ACCESS TO PLATFORMS THAT ALREADY EXIST AND HOPEFULLY OTHER PLATFORMS CAN, YOU KNOW, INTEGRATE INTO THIS ALSO. SO IF YOU LOOK ON THE NEXT SLIDE, AGAIN, WE'VE STARTED WITH SOME REAL TRAINING PILOT TRAINING PROJECTS, WHAT WE'RE DOING IS LITERATURE MINING, TO BRING THE INFORMATION WE COULD TOGETHER FOR THESE DISEASES. AND SO WE STARTED WITH CREATIN TRANSPORTER DEFICIENCY, AND WE DID A VERY MANUAL DETAILED CURATION OF LITERATURE AND BROUGHT IN ALL OF THE KNOWN VARIANT DATABASES. IF YOU SEE THERE ON THE RIGHT THERE'S THE PROTEIN, IT'S A TRANSMEMBRANE PROTEIN. AND ALL OF THE MUTATIONS ARE HIGHLIGHTED ON THE PROTEIN. YOU CAN SEE THE PROTEIN STRUCTURE WITH EACH OF THE MUTATIONS, AND YOU CAN ALSO -- IT'S HARD TO SEE, BUT IN THE BOTTOM RIGHT IS ACTUALLY THE ACTIVITY OF EACH OF THOSE MUTATIONS USING A CREATINE TRANSPORTER ASSAY INTEGRATED INTO THE FIRST INVESTIGATION OF THIS BIOINFORMATIC DATABASE. WHAT WE FOUND BY DOING ONE CASE STUDY IF YOU LOOK AT THE VARIANTS, ALL VARIANTS, YOU LOOKED AT THE PUBLIC DATABASES, THERE WAS NOT A GREAT DEAL OF OVERLAP, WHAT WAS IN ClinVar, ONE OF THE, YOU KNOW, VERY WELL KNOWN, WHERE PEOPLE PUT THEIR VARIANTS, WE CURATED 132. THERE WERE 406, ONLY 43 -- 53 OVERLAPS, ON PATHOGENIC, EVEN LESS, ONLY 18 OVERLAPS BETWEEN WHAT WAS CURATED IN LITERATURE VERSUS WHAT IS ACTUALLY IN ClinVar. IF YOU GO TO THE NEXT SLIDE, SO, AGAIN, WHAT I WANT TO POINT OUT, THIS IS A HIGHLY COLLABORATIVE EFFORT. AGAIN, WE'RE WORKING WITH NCI AT FREDERICK, NATIONAL CANCER INSTITUTE THE REASON WE WORKED WITH THEM IS THAT THEY HAVE A LOT OF TOOLS AND THEY ALREADY ARE INFRASTRUCTURE BUILT SO, AGAIN, WE'RE NOT TRYING TO REINVENT THE WHEEL AND BUILD SOMETHING NEW. WE'RE TRYING TO INTEGRATE AND SEE IF WE CAN INTEGRATE DATA INTO WHAT THEY HAVE ALREADY ESTABLISHED. SO WE'RE ALSO COLLABORATING WITH OTHER INSTITUTES, AS YOU CAN SEE, NICHD, AND NINDS, AND WE HOPE TO COLLABORATE WITH OTHER NIH INSTITUTES BECAUSE SOME OF THE DATA THAT WE WOULD REALLY LIKE TO HARNESS IS FROM THE NATURAL HISTORY STUDY THAT THE NIH CLINICAL CENTER IS ONE WAY WE CAN CONTRIBUTE IN NEW DATA INTO THIS DATABASE. SO IF YOU GO TO THE NEXT SLIDE. SO, THOSE WERE THE FIVE EXAMPLES THAT I HOPE SHOWED YOU, I GAVE A GOOD REPRESENTATION OF PRE-CLINICAL DEVELOPMENT PLATFORMS. AND HOW WE TRY TO ADD VALUE TO PROJECTS OF HIGH TRANSLATIONAL IMPACT. AND MOVE THINGS INTO THE CLINIC FOR TESTING AND NEW THERAPIES FOR PATIENTS. AND THIS IS A SNAPSHOT, OF OUTCOMES THAT WE THOUGHT, AGAIN, THIS IS MEASUREMENTS OF INDs AND PHASE 1, PHASE 2s, AND NATURAL HISTORY STUDIES THAT WE'VE SUPPORTED. AND SO, AGAIN, WE'VE HAD 44 INDs SINCE THE START OF THE PROGRAMS, AND THAT'S 32 INDs BECAUSE THE PROGRAM STARTED MANY -- SOME STARTED BEFORE NCATS WAS ESTABLISHED, AND CAME INTO NCATS, SO SINCE NCATS WAS ESTABLISHED THERE'S BEEN 32 INDs, SO, AGAIN, WE DON'T ALWAYS DO THE WHOLE IND. SOMETIMES IT'S THE PIECES THAT WE CONTRIBUTE, BRINGING PIECES TOGETHER. AND, AGAIN, THE CLINICAL TRIALS HAVE EITHER BEEN AT THE NIH CLINICAL CENTER IF FOR RARE DISEASES OR WITH COLLABORATORS, INITIATED BY WHO WE'VE COLLABORATED WITH. AND SO AS JONI SAID, WHAT WE WANTED TO HIGHLIGHT WAS THAT ONE OF OUR CONTRIBUTIONS TO A PROJECT HAS LED TO AN ACTUAL APPROVAL OF A DRUG, AND WHAT WE HOPE IN THE FUTURE AS JONI SAID OUR MANTRA IS DECADE AHEAD, MORE TREATMENTS FOR ALL PEOPLE, MORE QUICKLY, IF YOU GO TO THE NEXT SLIDE I THINK I'M JUST GOING TO END WITH THE APPROVAL, SO THIS WAS A FIRST FDA DRUG APPROVAL THAT CAME FROM A CONTRIBUTION THAT WE MADE TO A THERAPEUTIC DEVELOPMENT PROJECT. SO TDB WORKED ON A COLLABORATION ON NOVEL AZOLE CLASS OF ANTI-FUNGAL DRUG ON CRYPTOCOCCAL MENINGITIS, DID PRE-CLINICAL STUDIES. WHAT CAME OUT WAS A PLATFORM FOR THE MANUFACTURING WHERE IP WAS GENERATED, AND WE DEVELOPED CRITICAL PROCESS CHEMISTRY, ALL METHODS FOR LARGE SCALE MANUFACTURING, THAT WERE ALL LICENSED BY VIAMET, ACQUIRED BY MYCOVIA IN 2018, WERE ABLE TO APPLY CHEMISTRY METHODS TO APPROVAL. YOU CAN SEE ON RIGHT CURRENT CEO OF MYCOVIA COMMENTED THEY WERE GRATEFUL FOR THE COLLABORATIVE INTERACTIONS THEY HAD FOR THE PROCESS DEVELOPMENT FOR VH1 129 NOW, NOW VT-1161, FOR MUJOVA, FOR CHRONIC YEAST INFECTION. AGAIN, IT'S FOR WOMEN'S HEALTH. SO WE FEEL THAT, YOU KNOW, WE'VE HAD A GREAT VALUE ADDED, IN HOW WE WERE ABLE TO HELP IN THE DEVELOPMENT OF THIS DRUG. I'LL END THERE. I NEED TO THANK EVERYBODY BECAUSE THIS IS A TEAM EFFORT, LIKE EVERYONE IS SAYING. AND EVERYTHING WE DO IN OUR TDB PROGRAM IS A TEAM EFFORT WITH OUR COLLABORATORS, EVERYBODY HERE AT NIH, OUTSIDE OF NIH, AND ALL OF OUR FUNCTIONAL TEAMS THAT WORK TOGETHER ON ALL OF THE PROJECTS SO I WANT TO SEND OUT A REALLY BIG THANK YOU TO EVERYBODY THAT WE WORK ON ALL OF OUR PROGRAMS WITH. AND LASTLY I THINK IT'S JUST OPEN FOR DISCUSSION, IF ANYONE HAS ANY QUESTIONS. AND THANK YOU FOR LISTENING. >> LIZ, THANK YOU FOR THE TREMENDOUS OVERVIEW, REALLY TERRIFIC. I THINK WE HAVE A FEW OPPORTUNITIES TO HEAR SOME DISCUSSION OR QUESTIONS. FIRST I WANT TO ASK ANDREW, HE PUT INTO THE CHAT THE IDEA OF, WELL, THIS PAPER THAT WAS PUBLISHED RECENTLY, AND I WAS WONDERING, ANDREW, IF YOU WOULDN'T MIND SAYING A FEW WORDS ABOUT THAT MANY BECAUSE I THINK IT'S SORT OF HIGHLIGHTING THE IDEA OF WHAT WE DO PUTTING PIECES TOGETHER AND CONNECTING THE DOTS. >> SURE. WELL, THIS IS A CASE STUDY ABOUT THE AADC DEFICIENT INFANTS THAT NCATS PLAYED A CRITICAL ROLE PARTNERING WITH AGILLUS. IT'S A SINGLE GENE MUTATION THAT BASICALLY PREVENTS INFANTS FROM PRODUCING DOPAMINE IN THE BRAIN. MOST OF YOU KNOW FAR BETTER THAN I DO THAT WITHOUT DOPAMINE WE AREN'T ABLE TO ENGAGE IN MOTOR FUNCTIONS. AND THIS TAIWANESE DOCTOR, PAUL HU, DEVELOPED THE GENE THERAPY THAT ULTIMATELY WAS TAKEN FORWARD THROUGH COMBINATION OF GRANTS FROM NCATS AND AGILLUS IS, AND AFTER THE FDA SAW THE PHASE 2 DATA, THEY SAID YOU DON'T NEED TO DO A PHASE 3, YOU CAN APPLY FOR A BIOLOGICS LICENSE BECAUSE THE RESULTS WERE JUST SO DRAMATIC. INFANTS THAT COULDN'T MOVE, COULDN'T LIFT ARMS, COULDN'T SIT UP, COULDN'T MOVE AROUND, THEY ACTUALLY STARTED CRAWLING AND EVENTUALLY WALKING. REALLY KIND OF A MIRACULOUS THERAPEUTIC. AND THIS WOULD NOT HAVE HAPPENED WITHOUT NCATS. JUST A REALLY EXTRAORDINARY EXAMPLE OF WHAT YOU ALL HAVE ACCOMPLISHED OVER A VERY SHORT PERIOD OF TIME. >> THANKS, ANDREW. I APPRECIATE THAT. YEAH, HOPEFULLY WE CAN GET PAST THE FIREWALL SO THAT OTHERS CAN SEE THE PAPER AS WELL. APPRECIATE THAT. I SAW MARSHAL HAD A HAND UP EARLIER. >> GREAT PRESENTATION. I'VE BEEN WORKING WITH Y'ALL'S GROUP IN THE PAST. WE ACTUALLY DEVELOPED SOME RARE DISEASE DEVICES YEARS AGO WE LICENSED OUT, HOPEFULLY SOMEDAY THEY WILL COME TO A CLINIC NEAR YOU. BUT YOUR PROGRAM WHERE YOU'RE LOOKING AT CATEGORIZING AND STRUCTURING THE RARE DISEASE, I THINK THERE'S SOME GOLD IN THAT THERE MINE FROM THE STANDPOINT OF THOSE ARE THINGS THAT COULD ALSO BE ADAPTED FOR MOLECULAR VARIATION CHARACTERIZATION. YOU KNOW, KIND OF LIKE HOW WE USE KEG IN BIOCHEMICAL PATHWAYS TO TIE THINGS TOGETHER, USEFUL TO MOLECULAR GENETICS FIELD AS WELL, SO KEEP THAT IN MIND. >> GREAT. >> MATIAS? >> YEAH, CONGRATULATIONS. REALLY INSPIRING AND FASCINATING TO SEE WHERE YOU CAN FILL IN GAPS AND PUSH THINGS FORWARD. AND OBVIOUSLY WE HAVE MORE THAN 7,000 CASES WHO NEED YOUR HELP. I THINK IT'S A CHALLENGE OBVIOUSLY. WHAT I REALLY LOVE TO SEE AND LOVE TO HEAR FROM YOU HOW NCATS AND NIH AND THE COMMUNITY CAN BE MORE HELPFUL TO HELP YOU WHENEVER YOU GET TO A WALL YOU BREAK DOWN AN IMPEDIMENT FOR DEVELOPMENT, FINDING THE RIGHT PARTNER TO TAKE THE MOLECULE FORWARD WITH RESOURCES, FUNDING, TEAMS, WHO CAN OFFLOAD THE WORKLOAD YOU HAVE. WE HAVE A VERY VIABLE, VERY WELL RESOURCED BIOMEDICAL PRIVATE COMMUNITY WHO IS NOT ENGAGING IN SOME OF THESE PROBLEMS BECAUSE FOR THEM IT'S TOO RISK/BENEFIT RATIO NOT BENEFICIAL. AS YOU'RE DE-RISKING THAT OPPORTUNITIES BECOMES VIABLE AND CAN PUSH THINGS FORWARD INTO CLINICAL PIPELINES. AND ESTABLISHING PUBLIC/PRIVATE PARTNERSHIPS IS NON-TRIVIAL, I WOULD LOVE TO HEAR WHAT'S WORKING, AND WHERE SOME OF THE WALLS ARE IMPEDING. >> YEAH, THAT'S ALWAYS BEEN ONE OF OUR -- WE KEEP THAT IN OUR MIND. HOPEFULLY, I DON'T KNOW IF LILLY IS ON TOO, OR DON, WHEN WE'RE -- WE TRY TO THINK HOW DO WE GET THIS TO THE NEXT PARTNER, RIGHT? AND HOW DO WE -- WE'VE HAD SOME SUCCESS. YOU KNOW, A LOT OF -- WELL, SOME PROJECTS COME IN, THEY ACTUALLY HAVE A SMALL BIOTECH COLLABORATOR, BUT I THINK IT'S MORE IN THE ACADEMIC SPACE, RIGHT? WHEN PEOPLE COME IN AND THEN HOW DO WE HELP BRING THEM THE PARTNERS THEY NEED TO TAKE IT FORWARD, AND I THINK WE THINK ABOUT THIS A LOT ALSO IN THE GENE THERAPY SPACE BECAUSE OF THE SMALL NUMBER OF PATIENTS, HOW CAN THAT, YOU KNOW, WE'RE GOING TO TREAT THE PATIENTS THAT EXIST, YOU KNOW, CURRENTLY, BUT, AGAIN, IF IT'S A THERAPY THAT'S HELPFUL, HOW DOES THAT KEEP GOING, RIGHT? HOW DOES IT, YOU KNOW, BE ABLE TO HELP PATIENTS IN THE FUTURE. SO I THINK THOSE ARE ALL QUESTIONS THAT WE HAVE THOUGHT ABOUT, BUT I'M GOING TO SEE IF ANYONE -- I THINK WE'RE STILL AT THAT -- WE'VE BEEN FOCUSED MORE RIGHT NOW ON TRYING TO OPERATIONALIZE AND TO REALLY UNDERSTAND THE PATHWAY, BUT I THINK FOR THE N-of-1 AND SMALL DISEASES I THINK THIS IS A CRITICAL COMPONENT THAT WE THINK ABOUT AS THINGS GET MUCH MORE TOWARDS PRECISION MEDICINE ALSO. >> THIS IS DISEASE OF N-of-1, AND IT ISN'T, BECAUSE THERE'S A BILLION DOLLAR MARKET BEHIND THAT, BECAUSE JUST TALKING HERE, HYPER PARATHYROIDISM IS THE SCOURGE OF PEOPLE CAN END STAGE KIDNEY DISEASE, YOU'RE DESCRIBING EFFECTIVE STRATEGY TO BLOCK RECEPTOR WHICH IS CHRONICALLY ACTIVATED IN CHRONIC KIDNEY DISEASE AND END STAGE RENAL DISEASE, POTENTIALLY A HUGE DOWNSTREAM MARKET AVAILABLE, HOPEFULLY IF THAT IS NEGOTIATED YOU GETS SIGNIFICANT RETURN OF THAT INVESTMENT EMPOWERING YOU TO DO MORE WORK IN THAT SPACE SO REALLY STARTING AND CONTINUOUSLY REEVALUATING THE PROGRAMS YOU'RE MAKING AGAINST THESE OPPORTUNITIES TO OBVIOUSLY FOR THE APPROACH, GENE THERAPY, REALLY ENABLING AND EMPOWERING THE WORK BUT CONSTANTLY POKING THE PRIVATE SECTOR TO SEE WHICH OF YOUR PLATFORMS CAN BE LEVERAGED ABOVE AND BEYOND WHAT YOU DO, SOME INVESTMENT YOU CAN DO MORE GOOD GOING FORWARD. >> YEAH. AT ONE POINT, I DON'T KNOW HOW MANY YEARS AGO, MAYBE BACK IN 2013 OR 14, WE DID HAVE KIND OF A SHOWCASE, RIGHT? WE TRIED TO HAVE A MEETING WHERE WE TALKED ABOUT ALL OF OUR PROJECTS AND WE OPENED IT UP, IT WAS IN BOSTON ACTUALLY, TRYING TO SEE WHO MIGHT BE INTERESTED AND SO MAYBE WE HAVE TO DO SOMETHING LIKE THAT AGAIN, I THINK WE CALLED -- WE MADE PEOPLE AWARE OF WHAT WE'RE WORKING ON AND SEE IF THERE IS ANY INTEREST TO ACTUALLY FIND PARTNERS, LIKE YOU SAID, IF THERE ARE OTHER INDICATIONS WHERE, YOU KNOW, THERE COULD BE AN EXPANSION OF SOMETHING, WE'RE PROBABLY NOT AWARE EITHER. LIKE YOU SAID, THERE'S A LOT GOING ON IN CLINICAL INVESTIGATORS, WHAT THEY ARE DOING IN THE CLINIC, RIGHT, THAT COULD ACTUALLY HAVE APPLICATION. BUT MAYBE WE DON'T KNOW ABOUT EACH OTHER, RIGHT? >> AND COMMERCIAL LANDSCAPE HAS SIGNIFICANTLY ACCELERATED FOR RARE DISEASE, OVER THE LAST TEN YEARS. WE HAD FDA PANEL EARLIER THIS WEEK, WITH, YOU KNOW, NCATS, AND FDA WHERE I THINK I HEARD FROM TINA WE HAD OVER 900 ATTENDEES LOOKING AT MY LINK, THERE WAS A LOT OF INTEREST FOLLOWING FROM THOSE, IN ALSO SEEING COMMERCIALLY VIABLE STRATEGIES. SO DON'T UNDERESTIMATE THE POWER AND IMPACT YOU CAN HAVE IF YOU CAN PARTNER AND THERE BY, YOU KNOW, KEEP BREAKING DOWN THESE BOUNDARIES AND MAKING SOME OF THESE PROJECTS VIABLE FOR PRIVATE SECTOR AND I WAS HOPING THAT MARCUS WOULD UNMUTE AND JOIN BECAUSE IT'S VERY MUCH A A TOPIC CLOSE TO YOUR HEART. >> ELIZABETH, THIS IS MARSHALL, COME DOWN THE STREET TWO OR THREE MILES, WE'LL GIVE YOU A VENUE, LIVE CAST, AT OUR FACILITY. WE'VE GOT BEAUTIFUL AUDITORIUMS, ALSO AREAS YOU COULD WORK IN. LET US KNOW AND WE'LL HELP YOU SET IT UP. >> SOUNDS GREAT. THANK YOU. >> THANK YOU. >> GO AHEAD. >> WHAT WHY WERE YOU GOING TO SAY? >> YOU GO FIRST. >> THANK YOU FOR THAT PRESENTATION. THAT WAS HELPFUL TO SEE PRODUCTIVITY OVER TIME FOR THE DIVISION. ONE QUESTION THAT COMES TO MIND, OBVIOUSLY WE'VE ALL SEEN THIS METRIC PRESENTED IN TERMS OF THE COST OF DRUG DEVELOPMENT, I'M CURIOUS TO KNOW CUMULATIVELY HOW MUCHS THAT NCATS PUT INTO THE -- I'M NOT SAYING INTO ALL THE ACTIVITIES BUT ACTIVITIES YOU HAVE FUNDED, DO YOU HAVE AN ACCOUNTING OF HOW MUCH YOU'VE SPENT ON THESE THINGS THAT LEAD TO THE 44 INDs AND POTENTIAL FOR APPROVALS THAT MIGHT HAPPEN THIS YEAR? IS THERE A NUMBER? IT CAN BE A BALLPARK NUMBER, I'M NOT EXPECTING SOMETHING THAT HAS PRECISION. >> YEAH, I DON'T KNOW, AGAIN -- >> I SEEM TO ALWAYS GIVE TASKS TO NCATS THAT REQUIRES THEM TO DO MORE WORK OFF CYCLE LIKE WITH ADMINISTRATIVE SUPPLEMENTS, NOW ASKING FOR HOW MUCH MONEY DID YOU ACTUALLY SPEND ON THIS. BUT IF THERE IS A WAY TO UNDERSTAND THAT, I THINK THAT WOULD BE -- ANOTHER SELLING POINT, IF YOU WILL. THE WAY TO PROBABLY THINK ABOUT THAT IS WHATEVER YOU SPEND, I'M SURE IT'S NOT $2 BILLION, RIGHT? WHATEVER YOU SPENT, YOU CAN ESSENTIALLY POINT OUT IT'S CATALYTIC IN THE CONTEXT OF ESSENTIALLY THE OTHER MONIES IT HAS BROUGHT IN. THEREFORE HAS LED TO THIS. THAT'S THE NARRATIVE I WOULD BE INTERESTED IN SHOWCASING. >> YEAH, AND WE'RE SAYING -- I'LL JUST ADD TO THIS A COUPLE THINGS. ONE IS THAT WE'RE -- THIS IS SOMETHING WE'RE ACTIVELY LOOKING INTO. IT'S NOT AS EASY AS YOU MIGHT THINK IT IS. YOU PROBABLY DO THINK IT'S NOT EASY. >> I CAN APPRECIATE IT'S NOT EASY. I'VE OBVIOUSLY WORKED ON THE OTHER SIDE. IT'S NEVER EASY TO DO THE ACCOUNTING BUT IT IS SOMETHING THAT I THINK IS USEFUL WHEN YOU'RE MAKING THIS CASE. >> WE'RE ACTIVELY WORKING ON SOMETHING LIKE THIS, AND I DON'T THINK I CAN GIVE NECESSARILY A TIME WHEN WE'RE GOING TO BE COMPLETING WHAT WE'RE EVALUATING AT THE MOMENT BUT AT SOME POINT I HOPE WE CAN SHARE SOME OF THAT INFORMATION BACK WITH YOU. YEAH, WE'RE LOOKING INTO IT. DON, YOU PROBABLY HAVE SOMETHING TO ADD AS WELL. THE OTHER THING I WAS GOING TO ADD TO THE CONVERSATION WITHIN THE BGTC CONSORTIUM, GENE THERAPY SPACE, WE'RE GOING TO BE LOOKING REALLY HARD AT THE COMMERCIALIZATION POTENTIAL AND THE IDEA OF -- THE WAY WE'RE THINKING ABOUT THE DEMOCRATIZATION OF INFORMATION, BIOLOGY GENERATION, VECTOR GENERATION, AND I THINK THAT'S GOING TO ADD TO THIS VERY COMPLEX SPACE OF, YOU KNOW, HOW THAT'S GOING TO PLAY INTO THE COST SAVINGS IN TERMS OF DOING THIS KIND OF WORK. I JUST WANTED TO ADD THAT AS WELL. THAT I THINK WE'RE GOING LEARN A LOT FROM THAT PARTICULAR PROJECT TOO. DON, DO YOU HAVE ANYTHING TO ADD ABOUT THE IND? >> YEAH, I WAS GOING TO COMMENT MORE GENERALLY. INDEED IT'S A LITTLE HARD TO DECONVOLVE AND AGGREGATE COST, BUT CAN DEFINITELY GET SOME SENSE. BUT I THOUGHT I WOULD GIVE THE GENERAL BLANKET ANSWER CERTAINLY OUR DEVELOPMENT COSTS ARE NOT CHEAPER. AND THE GOVERNMENT DOESN'T REALLY WORK FASTER. AND SO IN A WAY WHEN WE LOOK BACK, RETROSPECTIVELY IT'S MORE ALONG THE LINES OF WHAT DR. KRETZLER WAS SAYING, WE'RE STUCK AT THIS OR THAT STAGE BECAUSE IT'S ANSWER ACADEMIC LAB, OR SMALL BIOTECH, SO IN A WAY ANOTHER QUESTION THAT CAME UP I THINK HAS TO DO WITH TIMING, OF OUR ROLE, OR THE FINANCIAL BENEFITS. ALONG WITH THAT IF WE LOOK BACK AT THE PORTFOLIO IN A WAY OUR BIGGEST IMPACT IS REALLY NEVER A SIDE-BY-SIDE SUBSTITUTE. SO IF SOMETHING COSTS $100 MILLION, WE PROVIDE IN-KIND SUPPORT FOR A COUPLE MILLION DOLLARS, THAT'S NOT THE VALUE. THE VALUE IS MORE OF WHAT LIZ IS SAYING, THAT WHATEVER THAT RISK PIECE IS, THAT IS NOT TENABLE IN THE COMMERCIAL SPACE OR FOUNDATION SPACE, BUT IT'S A RISK THAT WE'RE ABLE TO ABSORB, THAT'S THE MAJOR CONTRIBUTION. SO IN A WAY, THAT'S WHY WE AREN'T ALWAYS TALKING ABOUT DRUG PER DOLLARS BUT RATHER WHAT KIND OF RISK CAN WE ABSORB WHEN OUR ROI IS PUBLIC HEALTH, IT'S NOT ABOUT AN IMMEDIATE FINANCIAL RETURN. I THINK THAT DE-RISKING IS WHAT ALLOWS US TO HAVE A MAJOR IMPACT IN THE ECOSYSTEM AND TO HAVE SUCH A LARGE ACTUALLY HIGH SUCCESS RATE IN INDs, BECAUSE WE'RE ABLE IN TERMS OF NOT HAVING A COMMERCIAL ROI EXPECTATION TO STICK WITH THE PROJECT AND KEEP IT GOING AND WE ONLY FAIL A PROJECT WHEN IT'S SCIENTIFIC FAILURE, NOT BECAUSE WE'VE RUN OUT OF MONEY OR DON'T HAVE THE RIGHT TEAM ON BOARD, ET CETERA. ANYWAY, JUST TO VOLUNTEER MAYBE ANOTHER AXIS TO PLOT OUR WORK ON IS THE RISK AXIS AS OPPOSED TO MONEY PER SE. >> I DID WANT TO POINT OUT, I SAW LILLY PUT IN THE STRATEGIC ALLIANCE, THAT COLLABORATION AND PARTNERING HAS PLAYED A KEY ROLE LIKE IN ALL OF OUR PROJECTS, YOU KNOW, SO THAT'S A REALLY KEY COMPONENT THAT WE'VE ALWAYS WORKED VERY CLOSELY WITH LILLY'S GROUP IN ORDER TO THINK ABOUT THE PARTNERING ASPECTS AND COLLABORATION ASPECTS BECAUSE WE HAVE TO DO THAT THROUGHOUT A PROJECT AS WE'RE COLLABORATING WITH OTHERS. THERE'S A COUPLE OTHER HANDS UP. >> GO AHEAD. >> I JUST WANTED TO BUILD OFF OF THAT. ELIZABETH, I APPRECIATED YOU RECOGNIZING THE ROLES JANSEN FOUNDATION HAVE, ONE POWER IS POWER TO CONVENE AND BRING PARTNERS TOGETHER. BUILDING OFF THE LAST CONVERSATION WE HAD AROUND THE PUBLIC/PRIVATE PARTNERSHIPS AND HANDOFFS, THE MORE THAT NCATS CAN CONTINUE TO HELP PUT THESE CASE STUDIES TOGETHER AND HIGHLIGHT THE ROLE THAT PATIENTOS HELPED TO PLAY AS WE KNOW MANY TIMES THIS FUNDING AND INNOVATIONS START WITH PATIENT ADVOCACY ORGANIZATIONS, HELPING WITH EARLY FUNDING AND ADVOCACY THE FEDERAL GOVERNMENT OWN STARTS FUNDING STREAMS IN THESE DISEASE SPACES, AND IT'S REALLY IMPORTANT THAT WE CONTINUE TO CLOSE THAT FEEDBACK LOOP SO WE CAN MAKE SURE THAT EVERYBODY UNDERSTANDS WHAT HAPPENS AT THE END, FUNDING STARTING OFFED WITH THE ORGANIZATION, IF IT ENDS IN A THERAPY AFTER HANDOFFS WE DON'T LOSE THE THREAD THIS STARTED WITH EARLY ADVOCACY, CONVENING STAKEHOLDERS AND PARTNERS AND ACADEMICS AND CLINICIANS WHO MAY NOT HAVE COME TOGETHER WITHOUT THE FIRST PATIENT ADVOCACY MEETING, I APPRECIATE YOU HIGHLIGHTED THAT AS WE THINK ABOUT CONTINUING MESSAGING MAKING SURE WE DON'T LOSE THE THREAT. >> I TOTALLY AGREE. THAT'S SUPERPOWER ON QUITE A FEW PROJECTS, TALKED ABOUT WRITING UP OTHER CASE STUDIES WHERE THERE HAS BEEN A LOT OF POWER WHY PATIENT ADVOCACY, EVEN THE CREATINE TRANSPORTER I MENTIONED PROPELLED, LIKE NOBODY WAS WORKING IN THE FIELD, AND THEN A PATIENT ADVOCACY GROUP WAS FORMED AND ACTUALLY HAD THEIR FIRST SCIENTIFIC MEETING THAT BROUGHT TOGETHER DIFFERENT INDUSTRY CAME IN AND YOU NO THERE'S SEVERAL COMPANIES WORKING ON THE DISEASE SO AGAIN IT WAS A CATALYST THAT ALSO STARTED WITH WORKING WITH THE PATIENT GROUPS. >> I WANT TO SAY THESE CASE STUDIES THAT HAVE BEEN BROUGHT FORWARD, THEY HAVE JUST -- THEY HAVE MADE ME JUMP FOR JOY. THEY ARE THRILLING STORIES. I DON'T THINK THE GENERAL PUBLIC KNOWS THIS. THEY ARE CAPTIVATING. I DO THINK THERE'S AN OPPORTUNITY TO -- ROAD SHOW FOR PURPOSE OF HAVING MORE COLLABORATION IS GREAT BUT FROM MESSAGING TO THE GENERAL PUBLIC, I MEAN, REALLY COULDN'T BELIEVE THE STORY THAT WAS TOLD WHEN YOU ASKED FOR THE FEATURE ARTICLE SENT AROUND, THAT'S AN INCREDIBLE STORY. YOUR WHOLE PRESENTATION, ELIZABETH, WAS GREAT. I JUST WANT TO HEAR MORE. I DON'T THINK THE WORLD KNOWS. >> THANK YOU. >> YEAH, THANK YOU, KELLY. YEAH, WE'RE TRYING TO USE THE MEGA HORN, IS THAT WHAT THAT IS THAT YOU TALK INTO IN. >> RIGHT. >> TRYING TO GET THE MESSAGES OUT THERE. I AGREE, WE NEED TO DO MORE OF THAT. AND I HAD THE SAME REACTION WHEN LIZ WAS GIVING HER PRACTICE PRESENTATION, JUST SUPER EXCITED ABOUT THE WORK. AND RECOGNIZING THAT THESE ARE ALL CASES WE NEED TO SOMEHOW CREATE INTO VIGNETTES AND MAKE SURE THE PUBLIC IS AWARE THAT'S GOING ON. >> YEAH, VIGNETTE IS THE RIGHT WORD. >> YEAH. >> ANDREW, YOU HAVE YOUR HAND UP. >> YEAH, FOLLOWING UP ON THE RESPONSE DON GAVE ABOUT RATE OF RETURN OR SOME KIND OF MEASURE OF IMPACT ACCOUNT , AND I POSTED ABOUT A CASE STUDY IN 2016 LOOKING AT NCATS RARE DISEASE PORTFOLIO, IN A CASE STUDY PROVIDES SOME NUMBERS THAT RAHESH IS LOOKING FOR. WHAT WAS SAID BEFORE IS CONFIRMED THAT NCATS IS NOT NECESSARILY FASTER IN TERMS OF THE CLINICAL TRIALS THEY HAVE RUN OR WORK THAT'S LED TO THE INDs, BUT IT IS TRUE THEY HAVE HIGHER SUCCESS RATES BUT THERE'S ANOTHER MEASURE OF IMPACT, I KNOW THIS IS NOT SOMETHING THAT NCATS IS FOCUSED ON, BUT AS AN ECONOMIST IT IS SOMETHING I'M FOCUSED ON WHICH IS THE FINANCIAL IMPACT THAT NCATS HAS HAD. A COUPLE INTERESTING DATA POINTS THAT YOU MIGHT CONSIDER, ONE IS THAT IN THE AADC CASE STUDY AGILLUS IS BIOTHERAPEUTICS WAS COLLABORATING WITH NCATS, THE REASON I GOT INVOLVED WAS BECAUSE AGILLUS HAD A HARD TIME RAISING MONEY. I WAS INTRODUCED BY A MUTUAL ACQUAINTANCE TO AGILLUS, AND IN TALKING WITH THEM IT WAS PRETTY CLEAR THAT IF IT HADN'T BEEN FOR NCATS COLLABORATION, THEY WOULD NOT HAVE GOTTEN AS FAR AS THEY DID, AND JUST TO GIVE AN IDEA OF HOW FAR THEY DID GET, AFTER A FEW MONTHS PASSED, THE PHASE 2 READOUT, THEY WERE ACQUIRED BY PTC FOR APPROXIMATELY $900 MILLION. ONE THERAPY. AND TEN PATIENTS. $900 MILLION. SO THAT GIVES A LITTLE BIT OF A SENSE OF THE KIND OF IMPACT THAT NCATS HAS HAD, AND IN THE 2016 CASE STUDY THERE WAS A SIMILAR EXAMPLE WHERE ONE OF THE THINGS I WAS INTERESTED IN WAS WHAT ARE THE R01 OF AN NCATS PORTFOLIO, IF WE TOOK 28 RARE DISEASE THERAPEUTICS IN THE PORTFOLIO AT THAT TIME, WHAT WOULD THE ROI HAVE BEEN? WE TALKED TO VENTURE CAPITALISTS TO PROVIDE A BACK OF THE ENVELOPE VALUATION, THE PAPER WAS SUBMITTED AND REJECTED BY SCIENCE TRANSLATIONAL MEDICINE, THEY SAID THESE NUMBERS ARE MADE UP. AND FROM AN ECONOMIST POINT OF VIEW THAT'S WHAT WE DO. WE END UP MAKING UP THESE RESULTS BECAUSE THOSE ARE THE FOLKS ENDING UP PAYING FOR IT. TURNED OUT WHILE OUR PAPER WAS BEING REVIEWED, AFTER WE HAD GOTTEN BACK THAT REJECTION, TWO OF THE 28 PROJECTS THAT NCATS WAS INVOLVED IN, THEY WERE AFFILIATED WITH COMPANIES THAT WERE ACQUIRED BY BIG PHARMA. AND THE ACQUISITION PRICES AND DEAL TERMS WERE NOT DISCLOSED, BUT IF YOU LOOK AT THE STOCK PRICE REACTIONS, OF THOSE TWO COMPANIES IT ACQUIRED, THE TWO TARGETS, THE STOCK PRICE INCREASES IN THE ACQUIRER'S AMOUNTED TO $650 MILLION, ONE-DAY STOCK PRICE CHANGES. SOY ONCE WE RESUBMITTED THAT TO SCIENCE TRANSLATIONAL MEDICINE THEY THEN PUBLISHED THE PAPER. >> GREAT ANECDOTE. >> IT IS A GREAT ANECDOTE. THANK YOU FOR TELLING THE STORY. AND HOW WE CAN CONTINUE TO FOLLOW THAT SORT OF IDEA IS -- I WISH THERE WERE AN EASY WAY TO DO THAT. SO ANDREW MIGHT NEED TO PICK YOUR BRAIN A LITTLE BIT ABOUT THAT. >> JUST THE FACT HE TOLD A STORY ABOUT A SINGLE ASSET WITH TEN PATIENTS THAT EXITED FOR MORE THAN THE ANNUAL BUDGET OF NCATS IS ASTONISHING. THE NOTION THAT SOME OF THAT SHOULDN'T FLOW BACK TO NCATS IS TOUGH TO STOMACH, AS A TAXPAYER. I MEAN, YOU MAY NOT HAVE -- I UNDERSTAND THAT THERE ARE PROCESSES IN PLACE AND I KNOW THAT THEY TOOK THE RISK AND GREW IT, BUT THAT IS THE MISSION OF NCATS IN ACTION, BUT YOU GUYS ARE NOT GETTING THE FLOWBACK AND REVENUE. PERHAPS THAT'S FINE. BUT -- >> CAN I SAY SOMETHING IN THAT CONTEXT? >> IT'S ACTUALLY AS LONG AS THAT BECOMES PUBLIC KNOWLEDGE, AND FACILITATES AND ADVANCES AND INCREASES THE UPTAKE OF THE OUTPUT FROM THE OPERATION, THERE'S SIGNIFICANT RISK AND A REASON WHY THESE ASSETS ARE MOVED AT THAT AREA, AND I THINK THIS IS THE KEY PIECE, THE CONCEPT OR WHATEVER IS THE BEST VENUE TO DO THAT, IT'S CRITICAL THAT, YES, WHENEVER AN ASSET IS DE-RISKED, PRIVATE SECTOR CAN TAKE ON THE RISK AND WILL REMAIN SIGNIFICANT, THERE WILL BE SIGNIFICANT INVESTMENTS NEEDED WHICH NCATS OR THE PUBLIC SECTOR CANNOT STOMACH. THAT I THINK, THAT IS A SUCCESS MODEL. WHICH REALLY WORKS WELL, OBVIOUSLY RESOURCES HAVE INCREASED OVER THE LAST DECADE TO DO THAT IN THE PRIVATE SECTOR. IF YOU CAN FACILITATE THAT WOULD BE FULLY ALINED WITH WHAT I CAN SEE AS KEY MISSION POINT. >> JONI, YOU USED THAT GRAPHIC THE OTHER DAY WHICH YOU USED ONCE BEFORE WITH US WHICH I LOVE, WHICH IS YOUR METAPHOR OF A PIPELINE. AND ACCELERATING WHAT FLOWS THROUGH THAT PIPELINE. AND THAT PIPELINE IS A VALUE CHAIN. THESE ARE JUST REALLY CONCRETE VIGNETTES TOLD TODAY THAT ARE SHOWING THE TIGHTLY COUPLED RELATIONSHIP IN THAT VALUE CHAIN. AND THAT VALUE, I'M VERY INTERESTED IN THE ECONOMICS OF IT EVEN THOUGH I KNOW THAT'S NOT MOSTLY WHAT WE TALK ABOUT IN THESE MEETINGS. SO, I DON'T KNOW. I'D BE HAPPY TO SPEAK WITH ANYBODY MORE ABOUT THIS. I THINK THERE'S SOME REAL OPPORTUNITIES. I'D BE HAPPY TO HELP WITH A ROAD SHOW AS WELL. THERE'S JUST SO MUCH VALUE YOU GUYS ARE CREATING. IT'S REALLY -- I'M ON THE EDGE OF MY SEAT. >> YOU'RE ONTO SOMETHING HERE. >> IN THE EARLY DAYS OF SETTING UP NCATS I WORKED WITH VENTURE CAPITALISTS TO TALK ABOUT MONETIZING PIECE, THE IDEA YOU COULD FIND A WAY IF YOU DID FUND THESE THINGS THAT COMPONENT OF THAT REVENUE WOULD FLOW BACK TO WHATEVER INSTITUTE AT THAT TIME, IT HADN'T BEEN NAMED, LET'S SAY THE NIH, BACK TO THE GOVERNMENT. WE DID SOME SIMPLE BACK OF THE ENVELOPE CALCULATIONS, BECAUSE WHAT HAPPENS IS THAT THINK ABOUT WHAT THE NIH DOES, IT FUNDS WORK, MULTIPLE ACADEMIC INSTITUTIONS, STANFORD, M.I.T., UNIVERSITY OF IOWA, WHATEVER, GETS LICENSED TO A COMPANY, AND THE ROYALTY STREAM GOES BACK TO THE GRANTEE. BUT THE GRANTEE NEVER CHOOSES TO SEND ANY PORTION OF THAT BACK TO THE FUNDER BECAUSE THERE'S NO -- BECAUSE OF THE DOLE ACT, IT'S NOT WRITTEN INTO LAW, BUT THE THING WE WERE EXPLORING AT THAT BECAUSE ALL IDEAS WERE WELCOME TO PUT AN AMENDMENT, IF GRANTEES GOT THAT SOME PERCENTAGE OF ROYALTY INCOME WOULD COME BACK TO THE GOVERNMENT. IF WE DID THAT FOR ALL DRUGS LICENSED PATENTS FROM THE PATENT ESTATE FUNDED BY NIH WORK THE NIH WOULD BE EVERGREEN, NOT JUST NCATS. WE WOULDN'T NEED TO ASK THE TAXPAYER FOR ADDITIONAL APPROPRIATIONS. BECAUSE THE REVENUE WOULD BE GREATER THAN $30, $40 BILLION A YEAR. THAT WOULD COME BACK. BUT WHEN WE ACTUALLY DID SOME CONVERSATIONS WITH DEANS OF UNIVERSITIES, BECAUSE WE WANTED TO SEE, PRESSURE TEST THIS, IT WAS A CATEGORICAL NO. THERE'S NO WAY I'M PARTING WITH A PENNY, RIGHT? AND GIVING IT BACK TO THE NIH BECAUSE EVERYBODY WANTS TO KEEP THAT FOR THEIR LOCAL FUNDING, PUT UP A NEW BUILDING, WHATEVER. ADDITIONAL OFFICE SPACE, ET CETERA. SO, YOU KNOW, I THINK IT'S A VERY DIFFICULT THING TO NAVIGATE. THE GOODWILL PIECE AND FACT WE'RE DOING IT, ET CETERA, BUT IF YOU WANT TO MAKE THAT HAPPEN IT'S ABOUT THE MONEY, RIGHT? THE MONEY HAS TO COME BACK TO BE ABLE TO DO THAT. THE ONLY MECHANISM IS TO PROBABLY WRITE IT INTO LAW. SO IF SOMEBODY HERE IS POLICY MINDED, SOMEBODY ESSENTIALLY COULD COME UP WITH A CREATIVE TIME, RIGHT TIME IN HISTORY TO DO, THEN WE'LL CHANGE THE ENTIRE BUSINESS. >> SOUNDS EASY. >> YEP. >> CHAD? >> I WANT TO COMMENT. I THINK THE POLICY IS STILL THE SAME BUT SO WHEN I HAVE AN R01, AND I MAKE A PATENT, AS WAS COMMENTED TO, THE NIH HAS NO PART OF THAT. THEY HAVE GIVEN UP ALL RIGHTS OF THE PATENT TO THE UNIVERSITY. HOWEVER, WHEN I HAD MY NCATS GRANT, AND DISCOVERED SOME MOLECULES THROUGH NCATS, NCATS HOLDS ON TO THAT PATENT. SO THEY WERE A CO-INVENTOR, I BELIEVE THAT'S STILL THE POLICY. THERE IS A MECHANISM FOR MONEY TO COME BACK, IF NCATS IS INVOLVED IN THE PATENT DISCOVERY. FOR THESE EXAMPLES YOU GAVE I'M NOT SURE IF NCATS WAS INVOLVED IN GETTING NEXT GENERATION PATENTS, BUT I'VE ALSO BEEN IN A BLUEPRINT GRANT WHERE WE TOOK AN NCATS DISCOVERY, WHICH NCATS HAD PARTIAL PATENT CONNECTION, AND THEN WE DID THE NEXT GENERATION OF THAT, AND I BELIEVE THAT THE BLUEPRINT WAS NOT PART OF THAT PATENT. SO, -- >> THAT'S RIGHT. WHEN I SET UP THE BLUEPRINT PROGRAM WE DID NOT PUT THAT IN PLACE. ONE OF THE CHALLENGES WAS THAT PEOPLE WEREN'T NECESSARILY COMING TO THE NINDS AT THAT TIME TO REQUEST THAT MONEY IF THEY NEEDED TO SHARE, THE INTELLECTUAL PROPERTY WITH THE NIH. THE INTRAMURAL PART OF NCATS WAS ABLE TO DO THAT BUT IN THE EXTRAMURAL SIDE THAT'S VERY DIFFICULT TO DO. >> YEAH, ON A PERSONAL LEVEL BECAUSE OF THAT, THAT BLUEPRINT, YOU KNOW, WE WOULD HAVE EASILY GIVEN UP THAT RIGHT TO THE PATENT THAT THEY HELPED US DEVELOP. I THINK YOU HAVE A LOT OF LEVERAGE BECAUSE THE P.I., WE'RE DESPERATE FOR FUNDS TO TAKE IT TO THE NEXT LEVEL AND CAN GO TO DEANS, HEY, WE NEED THIS MONEY, WE'RE GOING TO GIVE UP A PART OF THE ROYALTIES, IF IT GOES DOWN THAT ROAD. SO THAT COULD BE ANOTHER OPTION, IF YOU USE THE P.I.s TO LEVERAGE THE DEANS TO GIVE A PART OF THAT PATENT BACK TO NIH. BUT JUST WANTED TO POINT OUT THE ODD DETAILS. >> THANK YOU. >> I'LL POINT OUT JUST AN EXPERIENCE WE HAD, SO ACTUALLY INTRODUCED US TO A TECHNOLOGY GROUP OVER AT UNIVERSITY OF MARYLAND, WE WENT ON TO DEVELOP TWO DEVICES WORKING WITH THEM, DID NOT RECEIVE MONEY FROM NIH BUT WHEN WE FILED A PATENT NIH DID INSIST ON A PERCENTAGE OF THE PATENT. SO SOMETIMES EVEN IF MONEY DOESN'T COME OUT, NIH DOES STILL GET A PERCENTAGE OF THOSE. WE DIDN'T BALK, IT WAS FINE WITH US. IT WAS INTERESTING, NOT BECAUSE IT WAS NOT OF -- BECAUSE IT WAS NOT A FUNDED PROJECT. >> IT WAS A CO-INVENTION IN THE INTRAMURAL PROGRAM. >> ACTUALLY, IT WASN'T. "THEY" DID THE INTRODUCTION. IT WE WERE FINE WITH THEM HAVING A PERCENTAGE, HAPPY TO TALK TO YOU ABOUT IT ANOTHER TIME. >> OKAY. >> ANDREW, YOUR HAND IS UP. YOU'RE ON MUTE. >> SORRY ABOUT THAT. . I WANTED TO RESPOND ABOUT THE POINT AND KELLY'S QUESTION ABOUT NCATS GETTING BACK SOME OF THE VALUE CREATED. TWO THINGS. ONE, THAT'S A VERY OF VERY COMPLICATED QUESTION BECAUSE IF NCATS WERE TO GET PAID FOR THE THINGS IT DOES, AND GET INVOLVED IN COMMERCIAL KINDS OF ARRANGEMENTS, I THINK THAT WOULD ACTUALLY CREATE ALL SORTS OF CONFLICTS THAT WOULD UNDERMINE THE NCATS MISSION. AND SO I THINK THERE IS AN IMPORTANT SEPARATION BETWEEN PUBLIC AND PRIVATE ACTIVITIES, AND THAT WAS WRITTEN INTO LAW FOR THAT REASON. HOWEVER, I THINK THERE IS AN IMPORTANT POINT ABOUT NCATS BEING ABLE TO GET A MUCH BIGGER FOOTPRINT IN TERMS OF IMPACT IF IT WERE ABLE TO COLLABORATE WITH THE PRIVATE SECTOR. AND TO RAHESH'S POINT, AFTER THE PAPER, THE CASE STUDY IN 2016 WAS PUBLISHED, THERE WAS AN NCATS HOSTED OPEN HOUSE FOR SOME OF THE POTENTIAL PRIVATE SECTOR INVESTORS THAT I HAD BEEN TALKING TO ABOUT NCATS AND POSSIBLY COLLABORATING WITH NCATS IN A SOMEWHAT MORE DIRECT FASHION. I BROUGHT THESE INDIVIDUALS DOWN TO D.C., FOR THIS MEETING, AT THE NCATS FACILITIES, AND THIS GROUP INCLUDED REPRESENTATIVES FROM THE VENTURE CAPITAL COMMUNITY, HEDGE FUND COMMUNITY, DRUG ROYALTY INVESTMENT COMPANIES, AND WITHIN 24 HOURS AFTER COMING BACK FROM THAT MEETING AT NCATS, ONE OF THE HEDGE FUNDS SENT CHRIS AUSTIN A LETTER OF INTENT SAYING THEY WOULD BE HAPPY TO INVEST $200 MILLION IF THEY COULD GET ACCESS TO SOME OF THE IP IN THE NCATS PORTFOLIO, THEY WOULD PROVIDE THEIR OWN FUNDING. AND CHRIS' REACTION WAS, WELL, WHAT DO WE DO WITH THIS? WE'RE NOT AUTHORIZED TO DO THIS KIND OF A DEAL. AND SO OVER THE COURSE OF THE FOLLOWING SEVERAL MONTHS CHRIS TALKED WITH A NUMBER OF STAKEHOLDERS WITHIN NIH, LILLY PORETILLO WAS INVOLVED, THE CONCLUSION WAS ALONG THE LINES YOU NEED AN ACT OF CONGRESS TO BE ABLE TO ALLOW NCATS TO ENGAGE IN THESE PUBLIC/PRIVATE PARTNERSHIPS AT WHICH POINT I DISCUSSED SOME OF THIS WITH CONGRESSMAN JUAN VARGAS, REPRESENTATIVE FROM SAN DIEGO, FROM -- YEAH, SAN DIEGO. HE'S VERY INTERESTED IN BIOTECH ISSUES BECAUSE OF COURSE SAN DIEGO HAS HAD A VIBRANT BIOTECH COMMUNITY. HE SPONSORED THE VARGAS ROONEY PETERS BUILD, THE RARE DISEASE BILL OF 2018 GIVING NIH AUTHORITY TO CREATE A PRO SHALL DID SHALL PUBLIC/PRIVATE PARTNERSHIP. THAT BILL NEVER GOT OFF THE GROUND BECAUSE OF INFIGHTING WITH REPUBLICANS AND DEMOCRATS BUT DID GET SOME TRACTION AND THEN THE PANDEMIC HIT, AND THAT PUT EVERYTHING ON HOLD. I THINK THERE'S AN OPPORTUNITY. >> IF I MIGHT -- ANDREW BROUGHT THIS UP, THE ORIGINAL DISCUSSIONS THAT I MENTIONED WE CONVENED BCs, ET CETERA, WE HAD ADDRESSED THIS ISSUE IN TERMS OF, YOU KNOW, HOW WOULD WE AVOID CONFLICT OF INTEREST. WE COULD TALK BIT OFFLINE, STRAIGHTFORWARD MECHANISMS WHERE THE MONEY DOESN'T HAVE TO COME BACK TO THE SOURCE FROM WHICH IT CAME, FOR EXAMPLE, TO AVOID CONFLICT OF INTEREST. IT GOES INTO A CENTRAL PART. THE COLOR OF MONEY GETS WASHED, DIFFERENT WAYS TO DO THIS. THAT WILL BE ABOVE BOARD AND CAN ACTUALLY MAKE IT HAPPEN. I DON'T THINK THE MECHANISMS ARE DIFFICULT. IT'S WILL POWER, THE DESIRE, THE POLITICAL WILL TO WANT TO DO IT, THAT REQUIRES CONSTITUENTS IN THEIR -- ONE CONGRESSMAN PUTTING A BILL THERE IS FINE, SAN DIEGO MIGHT WANT TO DO IT, BUT IF HARVARD HOSPITALS AND SAN FRANCISCO PLACES, STANFORDS OF THE WORLD SAY NO, WE DON'T WANT TO DO THAT, THE BOTTOM LINE WE WOULD HAVE TO GET THE ACADEMIC COMMUNITY OR DEANS OF THE ACADEMIC COMMUNITY TO APPRECIATE HOW IF THEY LEND SUPPORT THERE WOULD BE A WIN-WIN SOLUTION. THAT'S WHAT NEEDS TO BE STRUCTURED. >> CAN I SUGGEST THIS WOULD BE A GOOD TOPIC TO REVISIT IN THE NEXT FEW MONTHS. THE REASON BEING THAT I THINK A LOT OF THINGS HAVE CHANGED. IN PARTICULAR WHAT HAS CHANGED NOT SO MUCH ON ACADEMIC MEDICAL SIDE BUT RATHER IN THE INVESTMENT COMMUNITY IS THE NOTION OF IMPACT INVESTING. THAT'S A BUZZWORD THAT'S NOW BECOME MUCH, MUCH MORE IMPORTANT THAN IT WAS BACK IN 2018. AND I THINK THERE ARE NOW A LOT MORE WILLING INVESTORS, INSTITUTIONAL INVESTORS, MUTUAL FUND COMPLEXES, AND LARGE ORGANIZATIONS, THAT ARE INTERESTED IN SEEING THEIR MONEY NOT ONLY HAVE FINANCIAL RETURN BUT TO HAVE SOCIAL IMPACT AND I CAN'T THINK OF ANYTHING MORE IMPACTFUL THAN WHAT NCATS IS DOING. I THINK WE SHOULD REVISIT THAT. >> YEAH, I'M SITTING HERE THINKING TOO THAT BY REVISITING PERHAPS THIS IS SOMETHING WE NEED EVEN A WORK GROUP COUNCIL-BASED WORK GROUP TO BE CHEWING ON A VARIETY OF OPPORTUNITIES BUT ALSO THE BARRIERS TO REACH THOSE OPPORTUNITIES GIVEN WHERE WE ARE. THAT'S GOING TO TAKE A VARIETY OF US TO DIG DEEPER TO SEE WHAT SOME OPTIONS COULD BE. I AGREE I THINK THIS IS WORTH PURSUING BECAUSE I HAVE ALSO CONSIDERED THE IDEA THAT EVEN IF WE HAVE AN OPPORTUNITY FUND, AS WE POINTED TO, WHETHER OR NOT THAT MAKES US EVERGREEN IT CAN CERTAINLY HELP CREATE OPPORTUNITIES FOR A VARIETY OF OTHER ACTIVITIES TO BE DONE. AND THAT'S -- THAT WOULD BE REALLY, REALLY HELPFUL FOR A VARIETY OF REASONS. BUT MAYBE THERE ARE SOME PARAMETERS AROUND THAT, I DON'T KNOW. THAT WOULD MAKE IT EVEN MORE PALATABLE. HAVING A FOCUSED EFFORT TO TALK THROUGH A LOT OF THESE ISSUES MUCH MORE DEEPLY MIGHT -- WE MIGHT BE RIPE FOR THAT. SO STAY TUNED. AND WE CAN REVISIT THIS AS WELL OVER THE COMING MONTHS AND CERTAINLY AT THE NEXT COUNCIL TOO. ANY OTHER QUESTIONS AT THIS POINT? THAT WAS A GREAT, GREAT, GREAT DISCUSSION. THANK YOU. THORNY AND OPTIMISTIC. ALL RIGHT. LIZ, THANK YOU SO MUCH. YOU WERE ON THE HOT SEAT FOR A WHILE THERE. APPRECIATE EVERYTHING THERE. >> GREAT DISCUSSION. OKAY. BYE. >> THANK YOU. OKAY. ANNA, ARE THERE ANY UPDATES YOU NEWED TO BRING -- NEED TO BRING TO OUR ATTENTION? >> I DO NOT. WE'RE AT A POINT WE CAN ADJOURN AFTER YOU MAKE ANY PARTING COMMENTS YOU'D LIKE TO. >> OKAY. WELL, WE'RE NOT CLOSING OUT JUST YET. I'M NOT GOING TO USE THE GAVEL. AND I THINK THAT'S -- WE'LL LEAVE THE LIGHT ON FOR YOU TONIGHT AND WE'LL SEE YOU TOMORROW. WE WILL RECONVENE AT 1 P.M. SO PLEASE BE SURE TO DO THE CONNECTION AT 12:45 TO MAKE SURE YOU'RE IN AND WITH THAT I THINK WE CAN ADJOURN FOR THE DAY. THANK YOU FOR THE GREAT DISCUSSION. SEE YOU TOMORROW.