>>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.