NOW. WELCOME, EVERYONE, I WANT TO
GAVEL US IN TO THE 34TH MEETING
OF THE NATIONAL CENTER FOR
ADVANCING TRANSLATIONAL SCIENCES
ADVISORY COUNCIL. WELCOME. THANK
YOU FOR PARTICIPATING IN THE
MEETING TODAY THOSE HERE AND
THOSE WHO ARE ONLINE. I DON'T
HAVE A LOT TO START OUT WITH
HERE TODAY BUT I'M GOING TO TURN
IT OVER TO ANNA WHO WILL BRIEFLY
GO THROUGH SOME OF THE LOGISTICS
AND CONFIRM FUTURE MEETING DATES
AND DO ROLL CALL. WE WILL DO
THAT FIRST THEN START.
>> ALL RIGHT. TODAY MEMBERS IN
ATTENDANCE ARE PAUL HARRIS.
ANNIE KENNEDY. MATTHIAS
KRETZLER.
>> PRESENT.
>> ROBIN MERMELSTEIN. KEITH
MUELLER. PAULA SHIREMAN.
>> HERE. ANNICA WAYMAN. WE HAVE
NO AD HOC MEMBERS PARTICIPATING
TODAY. NEXT.
>> KELLY MCVEARY.
>> I DIDN'T SAY YOU?
>> NO.
>> I'M SORRY, KELLY. KELLY
MCVEARY IS JOINING US. ALL
RIGHT. SO AS YOU CAN SEE OUR
AGENDA TODAY INCLUDES SOME
ANNOUNCEMENT, OUR DIRECTORS
REPORT. WE HAVE A GUEST SPEAKER,
DR. RENEE WEGRZYN, DIRECTOR OF
ARPAH WILL JOIN US THEN A BREAK
AND CLEAR ANSWER OF A CONCEPT
FOLLOWED BY TWO PROGRAM UPDATES
ONE FROM DIVISION OF CLINICAL
INNOVATION, ONE FROM THE OFFICE
OF DRUG DEVELOPMENT AND
PARTNERSHIP PROGRAMS AND THOSE
ARE THE UPDATES THEN TWO CONCEPT
CLEARANCE PRESENTATIONS. AFTER
THAT WE WILL ADJOURN FOR TODAY,
APPROXIMATELY 5, 5:30 THIS
AFTERNOON. NEXT SLIDE. SO
ANNOUNCEMENTS. BECAUSE WE ARE
MEETING IN PERSON THIS ROOM IS
NOT -- EXCUSE ME. BECAUSE WE ARE
MEETING IN PERSON WE ARE NOT
LOGGING IN TO THE ZOOM
INDIVIDUALLY. THE ROOM ITSELF IS
LOGGED IN TO THE ZOOM MEETING.
THOSE WHO ARE ATTENDING
VIRTUALLY OF COURSE ARE
PARTICIPATING AS ZOOM
PARTICIPANTS. FOR THIS REASON IT
IS IMPORTANT TO TURN YOUR TABLE
MICROPHONE ON AND REFRAIN FROM
MOVING IT TO AVOID ADDITIONAL
NOISE. WHEN YOU ARE SPEAKING TO
ENSURE YOUR COMMENTS CAN BE
HEARD BY THOSE WHO ARE CONNECTED
VIRTUALLY. IF YOU ARE
PARTICIPATING VIRTUALLY RAISE
YOUR HAND OR USE THE CHAT
FEATURE THEN WAIT UNTIL YOU ARE
CALLED TO PROVIDE INPUT. ALL
PARTICIPANTS PLEASE ALLOW
SPEAKERS TO MOVE THROUGH THEIR
PRESENTATION, NOTING YOUR
COMMENTS AND/OR QUESTIONS AND
PRESENTING THEM WHEN CALLED
UPON. VIRTUAL PARTICIPANTS MUST
ENSURE DURING THE MEETINGS
DISCUSSIONS OF CONFIDENTIAL
MATTERS ARE NOT INADVERTENTLY
RECORDED OR OVERHEARD BY
DISABLING MICROPHONE RECORDING
FEATURE ON VOICE ENABLED
DEVICES. THAT OCCURRED AND IS
PROBLEMATIC. ANNOUNCEMENTS
CONTINUED. WE ARE USING THE
VIDEOCAST FEATURE TODAY SO
AUDIENCE MEMBERS MAY SUBMIT
COMMENTS UNTIL OCTOBER 14TH BY
USING THE EMAIL FUNCTION
AVAILABLE ON THE NIH VIDEOCAST
SITE. USING THE FEEDBACK BUTTON
OR SENDING EMAIL TO NCATS
COUNCIL INPUT@MAIL.NIH.GOV. WHEN
YOU ARE SPEAKING YOUR CAMERA IS
ON TO SPOTLIGHT FOR REVIEW AS
VIDEOCAST. IF YOU ARE NOT ON
CAMERA, THE PREVIOUS SPEAKER
REMAINS SPOT LIGHTED. BACK TO
THE PREVIOUS SLIDE, I MISSPOKE
ON THE LAST BULLET. THIS IS
OPEN MEETING SO NO ISSUES IF
THERE'S ANYTHING DURING THIS
DISCUSSION THAT IS OVERHEARD. SO
THAT IS A CORRECTION FOR THE
RECORD. NEXT SLIDE. SO THE
FUTURE MEETING DATES FOR NCATS
ADVISORY COUNCIL AS OF NOW ARE
JANUARY 18 AND 19 AND JANUARY 30
AND 31ST IN 2024 AND 2025
RESPECTIVELY. THE PLAN IS FOR
JANUARY MEETINGS FOB VIRTUAL. IN
PERSON MEETINGS IN 2024 ARE
EXPECTED TO OCCUR MAY 23 AND
SEPTEMBER 26 AND 2025 AND MAY 22
AND SEPTEMBER 25TH. SO WITH
THIS, I WILL ASK OUR DIRECTOR TO
GO UP TO THE LECTERN AND DELIVER
THE REPORT TO THE COUNCIL.
>> ALL RIGHT. THANK YOU, ANNA.
DO SOME PLUGGING TO MAKE SURE
EVERYTHING IS WORKING FINE.
THANK YOU ALL, I HAVE A VARIETY
OF UPDATES TODAY. IT WAS --
THIS ROUND I'M TALKING MORE
ABOUT INITIATIVE DEVELOPMENTS
AND IDEAS AND SOME THINGS WE
HAVE BEEN DOING OF LATE AND I
HAVEN'T SHOWN AS MUCH DATA AS I
USUALLY DO BUT HOPE THAT YOU
WILL HAVE A GOOD SENSE OF WHAT
WE HAVE BEEN UP TO AND KIND OF
THE AREAS THAT WE ARE MOVING
INTO AS WELL. SO I WILL ADVANCE.
I HAVE BEEN TAKING TO THIS IDEA
OF GIVING YOU A BIT OF A SUMMARY
FROM WHAT WE TALKED ABOUT LAST
TIME. JUST TO REMIND ME TO MAKE
SURE I HAVE COMMUNICATED,
THOUGHT I COMMUNICATED LAST TIME
BUT HOPEFULLY JUST JOGS YOUR
MEMORY WHAT WE DID TALK ABOUT.
SO LAST TIME WE PRESENTED A
VARIETY OF UPDATES ON COVID FOR
EXAMPLE, AND SOME OF THE
ANALYSIS THAT WE HAD ON PAXLOVID
USE AND REAL DATA OF COVID-19
THERAPEUTICS. AND THAT WAS
REALLY THE FIRST INDICATION OF
END OF THE PANDEMIC AUTHORITIES
THAT WE HAD. SO I WILL BE
SPEAKING MUCH LESS ABOUT COVID
THIS TIME, A LITTLE MORE ON
OTHER INITIATIVES WE ARE
STARTING TO PULL THROUGH THE
THREAD THERE. AND I ALSO
MENTIONED A LOT ABOUT THERE WAS
A LOT OF DISCUSSION ABOUT THE
GENE THERAPY PROGRAMS BUT THE
BULK OF CONVERSATION LAST TIME
WAS AROUND THE STRATEGIC
PLANNING PROCESS. AND THOSE
DISCUSSIONS THERE IN. AND WE
WERE SORT OF EARLY INTERIM AND I
WOULD SAY NOW WE ARE LATE
INTERIM. SOLID MORE UPDATE ON
THAT FROM THERE. I LOOK FORWARD
TO YOUR COMMENTS BUT THINGS ARE
NOW A LITTLE MORE OFFICIAL AS I
WILL TALK ABOUT IN A LITTLE BIT
SO TODAY I WANT TO DIVE IN SOME
NEWS AN ANNOUNCEMENTS THAT WE
TYPICALLY DO AT BEGINNING OF
DIRECTORS REPORTS. THIS SLIDE IS
THE SAME SLIDE I SHOWED BACK IN
MAY, STILL SAME STATUS BUT MORE
I THINK UPDATE TO THIS
PARTICULAR ANNOUNCEMENT NOW IS
THERE WAS A STAT ARTICLE WITH
BERNIE SANDERS SAYING THAT HE
WOULD HAVE A NOMINATION HEARING
IN THE NEXT MONTH. SO THAT IS
WHAT WE UNDERSTAND MAY HAPPEN.
AS THINGS ARE FLEXIBLE SEE HOW
THINGS GO BUT HOPEFULLY STAY
TRUE BUT WE ARE HOPEFUL THIS
WILL START TO MOVE FORWARD.
UNTIL THEN, THINGS ARE IN THE
CAPABLE HANDS OF DR. LARRY
TABAK, ACTING DIRECTOR OF NIH.
STAY TUNED ON THIS. MEANWHILE
COUPLE OF OTHER UPDATES GOING ON
AT THE NIH AS WELL. SO WANT TO
GIVE A SENSE OF THIS. WE HAVE A
VARIETY OF OPEN POSITIONS
DENOTED AS CIRCLES IN RED. AND
SOME OF THE POSITIONS FILLED
THAT ARE NOW IN GREEN. WITHIN
OUR OFFICE OF DIRECTOR THERE'S A
VARIETY OF LEADERSHIP POSITIONS
THAT I DON'T LIST, QUITE A
NUMBER OF THEM. BUT THERE HAS
BEEN A FEW RECENT FIELD
POSITIONS IN THOSE LEADERSHIP
ROLES IN OFFICE OF DIRECTOR. THE
TRIBAL HEALTH RESEARCH OFFICE
WITH KARINA WALTERS. OFFICE OF
BEHAVIORAL SOCIAL SCIENCES WITH
JANE SIMONI AND LEGISLATIVE
POLICY ANALYSIS DIRECTOR WITH
KATE (INAUDIBLE) AND ALSO THE
OFFICE OF NUTRITIONAL RESEARCH
WITH THE DIRECTOR ANDREW
BREMMER. THOSE ARE SOME
POSITIONS STARTING TO BE FILLED
AND THERE'S A FEW MORE IN OFFS
IF OF DIRECTOR HOPE TO UPDATE
YOU ON NEXT TIME WE SPEAK. ALSO
MEANTIME VERY RECENTLY DR. PATTY
BRENNAN, A DEAR COLLEAGUE AND
FRIEND HAS ANNOUNCED HER
RETIREMENT FROM FEDERAL SERVICE
AS NLM DIRECTOR, HAS BEEN THE
DIRECTOR FOR SEVEN YEARS AND IS
RETIRING THE END OF THIS WEE WE.
SO WILL MISS HER PARTNERSHIP,
WORKING CLOSELY IN AREAS OF DATA
SCIENCE AND INFORMATICS AND
THAT'S NEAR AND DEAR TO OUR
HEARTS TOO. SO I LOOK FORWARD TO
SEEING HOW THIS PROCESS UNFOLDS
AS WELL. THEN ANOTHER AREA OF
BIG NEWS, WE HAVE ONE OF THE KEY
POSITIONS FILLED WITH THE NIAID
DIRECTOR NATIONAL INSTITUTE ON
ALLERGY INFECTIOUS DISEASE WITH
GENE MORATSO (PHONETIC) WE LOOK
FORWARD TO HER JOINING US THIS
FALL AT THE NIH. SO WE ARE
GETTING THINGS ROUNDED OUT WITH
PATTY REFIRING WE HAVE MORE TO
GO. AS ALWAYS SEEMS TO HAPPEN
THAT WAY. WE ARE EXCITED WITH
THE NEW LEADERSHIP WE ARE
BRINGING TO NIH. IN THE SAME
VEIN WE HAVE VICTIM AREAS HERE
IN TERMS OF HIRING AT THE NCATS.
LAST TIME I SHOWED YOU A BUNCH
OF RED BOXES AND THAT MEANT WE
HAD POSITIONS AS OPEN POSITIONS.
BUT NOW WE ARE STARTING TO FILL
THOSE AND WE ARE STARTING TO
TURN THOSE GREEN. SO YOU KNOW
MOST OF THESE ALREADY. BECAUSE
WE MADE PUBLIC ANNOUNCEMENTS
ABOUT THEM BUT HIGHLIGHTING NOW
FIRST AND FOREMOST IS DR.
DOMINIQUE KASHARD (PHONETIC)
SINCE 2017 HE SERVED PASS CHIEF
SCIENCE OFFICER AT THE
INTERNATIONAL SYNDROME
FOUNDATION. A PHYSICIAN, BY
TRAINING AND SHE HAS BEEN IN THE
PRE-CLINICAL AND CLINICAL
RESEARCH SPACE FOR A WHILE. AND
SHE'S ALSO RARE DISEASE
ADVOCATE. SO WE ARE EXCITED TO
HAVE DR. PICHARD HERE AND SHE IS
NOW I THINK TWO OR THREE WEEKS
IN. SO VERY EARLY IN HER TENURE,
WE ARE DELIGHTED SHE IS HERE. OF
COURSE I WOULD BE REMISS IF I
DIDN'T SAY MANY THANKS TO PJ
BROOKS WHO SERVED VERY CAPABLY
AS THE ACTING DIRECTOR IN THIS
ROLE AND ALSO ALL OF THE STAFF
IN DRDRI FOR EXPERTLY COVERING
ALL OF THE THINGS THAT CAME OUR
WAY OVER THIS TIME. IT IS A
WONDERFUL TEAM TO BE WORKING
WITH AND I'M EXCITED TO HAVE DR.
PICHARD AT THE HELM. ANOTHER
ROLE, NEW ROLE TO NCATS. WE HAVE
A COUNCIL THAT IS HELPING US
THINK THROUGH DIVERSITY EQUITY
INCLUSION ISSUES AND HEALTH
EQUITY ISSUESFUL ONE OF THE KEY
RECOMMENDATIONS THEY HAVE GIVEN
OVER THE COURSE OF THE TIME THEY
HAVE BEEN CONVENING IS WE SHOULD
BE THINKING ABOUT A SCIENTIFIC
DIVERSITY OFFICER FOR NCATS SO
WE TOOK THAT TO HEART. AND WE
WERE DELIGHTED NOW TO ANNOUNCE
FOR THE FIRST TIME DR. JEANITA
PRICHETT, NCATS FIRST SCIENTIFIC
DIVERSITY OFFICER ON OCTOBER
10TH. SHE BRINGS OVER A DECADE
OF GOVERNMENT EXPERIENCE AT THE
NATIONAL INSTITUTE OF STANDARDS
AND TECHNOLOGY AND SHE'S BEEN
WORKING AS A BENCH RESEARCHER IN
CHEMISTRY, AND SHE HAS BEEN AN
ACADEMIC PROGRAMS AND AS A
PROGRAM MANAGER MOST RECENTLY
AND SHE HAS ALSO BEEN LEADER OF
THE NIST OR INSTITUTE OF
STANDARDS AND TECHNOLOGY
DIVERSITY EQUITY AND INCLUSION
AND ACCESSIBILITY EFFORTS FOR
VARIETY OF YEARS THERE. MOST
RECENTLY. SO WE ARE EXCITED TO
HAVE HER BRING THAT EXPERTISE
AND EXPERIENCE TO NCATS WHILE
SHE WILL HELP US REALLY DEVELOP
THIS PROGRAM EVEN MORE AND WORK
CLOSELY WITH THE LEADERSHIP AT
NCATS TO CONTINUE WORK WE ARE
DOING IN THE DEI SPACE AND
HEALTH EQUITY SPACE. I LOOK
FORWARD TO TELLING YOU MORE
ABOUT THIS ROLE AS IT STARTS TO
UNFOLD AS WELL. LASTLY, YOU KNOW
BALKY (PHONETIC) WELL ALREADY
BUT THIS IS THE FIRST
OPPORTUNITY TO SHOW HIM AS
OFFICIAL DIRECTOR OF OFFICE OF
STRATEGIC ALLIANCES. BALKY HAS
BEEN AN NCATS SINCE 2011
BRINGING LEADERSHIP TECHNOLOGY
DEVELOPMENT AND EXPERTISE AND
HE'S GAINED EXPERTISE FROM
WORKING IN THE INDUSTRY SECTOR
AS WELL AS THE GOVERNMENT SECTOR
ACROSS THE NIH. HE SERVED AS THE
OSA'S DEPUTY DIRECTOR FOR MANY
YEARS AND SERVE AS ACTING
DIRECTOR, NOW THE DIRECTOR OF
THIS -- IN THIS ROLE. CONGRATS
TO ALL THREE OF THESE
INDIVIDUALS AND I'M EXCITED TO
HAVE OUR LEADERSHIP STARTING TO
BE ROUNDED OUT. THERE WERE THREE
OTHER RED BOXES UP THERE AND I
HOPE TO TALK TO YOU ABOUT THOSE
LATER ON AT THE NEXT COUNCIL
MEETING BUT WE ARE WORKING
DILIGENTLY TO FILL THOSE AS
WELCH A NOTE ON OUR BUDGET. THIS
THE BUDGET OUTLOOK AT A GLANCE,
A CHART I SHOW EVERY TIME TO
GIVE YOU A SENSE OF WHERE WE
ARE. THIS IS THE SAME EXACT
SLIDE I SHOWED IN MAY BECAUSE
REALLY NOTHING CHANGED TO DATE.
BUT THE -- YOU CAN SEE IN A
SENSE NCATS HAS TWO BUCKETS OF
FUNDING STREAMS AND THE CLINICAL
AND TRANSLATIONAL SCIENCE AWARDS
PROGRAM IS 68% OF THE PIE. ALL
OTHER OF THE NCATS ACTIVITIES
ARE ABOUT 32% OF THAT PIE. WE
ARE VERY GRATEFUL TO KNOW WE
HAVE BEEN ON A TRAJECTORY OF
INCREASING THAT PIE OVER LAST
COUPLE OF YEARS. SO IN 2023,
LAST YEAR WE HAD $923.3 MILLION
TO WORK IN THIS SPACE. THE
OUTLOOK FOR FY 24 IS THE
PRESIDENT PROPOSED A FLAT BUDGET
FOR 24 AND FOR NCATS
SPECIFICALLY THE HOUSE AND
SENATE HAVE PROPOSED THE SAME
FLAT BUDGET FOR US. AND SO WE
ARE CONTINUING TO WAIT AND SEE
HOW THESE BILLS RESOLVE. AND WE
WILL CERTAINLY KEEP YOU UPDATED
WHEN WE KNOW MORE. BUT AS WE
SPEAK WE ARE PREPARING FOR ALL
SORTS OF SCENARIOS IN CASE THERE
IS A NEED FOR PREPARING FOR
TIGHT FUNDING IN 2024, WE WILL
BE PREPARED FOR THAT AS WELL.
SOME OF THESE ACTIVITIES WE HAVE
DONE RECENTLY HAVE BEEN ALSO
AROUND OPPORTUNITIES TO SPEAK TO
CONGRESSIONAL COLLEAGUES ON THE
HILL DOING BRIEFINGS AND
ENGAGEMENTS ON THE HILL TO TALK
ABOUT WHO NCATS IS, WHAT WE DO,
AND HERE I'M SHOWING A FEW
THINGS, THESE ARE TISSUE CHIPS
WE HAVE I WAS ABLE TAKE ON A
ROADSHOW IF YOU WILL TO GIVE A
SENSE WHAT THE CHIPS ARE ABOUT,
WHAT THEY LOOK LIKE AND ALWAYS
IS SOMETHING THAT ENGENDERS
EXCITEMENT ABOUT WHAT WE DO AT
NCATS SO IMPORTANT TO GET THAT
MESSAGE OUT THERE. ALSO A GOOD
OPPORTUNITY TO GET FACES TO
NAMES AND DEVELOP RELATIONSHIPS
FOR THESE SORTS OF ACTIVITIES
THAT WE DO IN CASE THERE ARE ANY
QUESTIONS THAT COME ALONG OUR
WAY WE ARE ABLE TO PANS
QUESTIONS RELATED TO CLINICAL
AND TRANSLATIONAL SCIENCE FOR
OUR HILL COLLEAGUES. THEN ON THE
OTHER SIDE OF THE EQUATION ON A
VERY DIFFERENT NOTE,
SERENDIPITOUSLY I RAN INTO
SENATOR BOASEMAN ON ANOTHER
EVENT, HE GRACIOUSLY INVITED ME
TO SPEAK TO HIM AND HIS STAFFERS
IN OUR OFFICE SO I WAS ABLE TO
GO THERE AND TALK ABOUT CTSA
PROGRAM IN, A A ARKANSAS AND WOK
WITH FDA ON THE
MICROPHYSIOLOGICAL SYSTEMS AND
TISSUE CHIP WORK WE BECAUSE THAT
WORK IS CENTERED IN ARKANSAS SO
WE WERE ABLE TO TALK A LITTLE
BIT ABOUT THAT WORK. SO I JUST
WANTED TO SHOW THAT AS WELL. SO
VARIETY OF ACTIVITIES ALWAYS
ONGOING IN THAT SPACE. AND I
LIKE TO MAKE SURE THAT PEOPLE
ARE AWARE THAT WE HAVE THOSE
OPPORTUNITIES ON SOME OCCASIONS
TO DO THAT. ANOTHER ACTIVITY WE
HAVE BEEN DOING IS A
RECONSTRUCTION OF OUR WEB PAGE.
YOU WON'T KNOW THIS YET BECAUSE
IT IS NOT UP YET BUT WILL BE
COMING UP THE NEXT MONTH OR SO.
THIS WILL -- OUR NEW WEBSITE
WILL HAVE A NEW LOOK, A NEW
FEEL, AND I HOPE YOU WILL AGREE
IT IS SORT OF A REALLY
REFRESHING LOOK AND FEEL. EMILY
MARTY AND HER TEAM AND OUR
COMMUNICATIONS OFFICE WORKED
HARD TO DO A DEEP DIVE AND
ENGAGING THE NCATS COMMUNITY AND
STAKEHOLDERS TO SAY HEY, WHAT DO
YOU WANT TO SEE ON THE NCATS
WEBSITE? AND TAKE ALL THAT TO
HEART TO DEVELOP THIS MIGHT LOOK
LIKE SO WE HAVE NEW SECTIONS ON
IMPACT AND NEW SECTIONS ON
RESOURCES SORT OF TAILORED TO
AUDIENCES IN NEED. I HOPE THAT
WILL BE EASIER TO NAVIGATE FOR
PEOPLE WHO ENCOUNTER OUR
WEBSITE. WE ALSO WILL BE
PUTTING IN A WAY TO FILTER ALL
SORTS OF RESEARCH ACTIVITIES.
FUNDING AND NEWS AND THINGS OF
THAT NATURE TOO. AND SO WE HOPE
THIS WILL COME ACROSS AS A
REFRESHED LOOK. BUT WILL ALSO
HAVE THE ABILITY TO REALLY MAKE
SURE THESE ARE THE LATEST AND
GREATEST STATEMENTS WHAT NCATS
IS DOING NEW INITIATIVES WE ARE
DOING AND I'M EXCITED TO SEE
THIS TAKE HOLD. SO AGAIN, STAY
TUNED, WE ARE EXPECTING THIS
NEXT MONTH MAYBE A LITTLE LONGER
BUT SHOULD BE OUT RELATIVELY
SHORTLY. NOW ON TO ACTIVITIES
AND IMPACTS ACROSS THE
TRANSLATIONAL SCIENCE PIPELINE.
I KNOW I WOULDN'T SAY TOO MUCH
ABOUT COVID BUT ONE THING THEN
MOVE ON TO VARIETY OTHER
ACTIVITIES AS WELL. SO THE
REASON I WANT TO HIGHLIGHT COVID
IS BECAUSE WE ARE STARTING OUR
LAST ACTIVE 6 ARM OF THE
DECENTRALIZED CLINICAL TRIAL.
THIS IS A METAPHORMAN ARM THAT
IS NOW OPEN FOR ENROLLMENT. WE
EXPECT THIS TO HAPPEN OVER NEXT
SIX OR SO MONTHS FOR THAT
ENROLLMENT TO HAPPEN AND I WILL
UPDATE YOU OF COURSE AS WE
CONTINUE TO MOVE ALONG THAT
PROCESS. BUT IT ALSO GIVES ME AN
OPPORTUNITY TO HIGHLIGHT A FEW
OTHER THINGS RELATED TO COVID IN
TERMS OF IMPACT. USUALLY IN THIS
PARTICULAR TIME OF THE YEAR I
TALK ABOUT SOME AWARDS, USUALLY
FROM THE EXTRAMURAL COMMUNITY
BUT TODAY I HAVE THE HONOR AND
PRIVILEGE OF FOCUSING ON OUR
NCATS COMMUNITY AND I'M
DELIGHTED TO SAY BECAUSE OF THE
WORK THAT SARA DUNS MOORE HAD
DONE ON CLINICAL TRIALS AND
ACTIVE 6 SHE WAS RECOGNIZE AND
RECEIVED SECRETARY'S
COMMENDATION FOR THE EXCEPTIONAL
SERVICE FOR WORK DONE IN THIS
SPACE. SHE WILL TELL YOU SHE IS
NOT ALONE IN THIS, THIS IS A
TEAM EFFORT BUT FORTUNATELY WE
WERE ABLE TO RECOGNIZE HER FOR
THIS EFFORT TO REPRESENT THAT
WHOLE TEAM. SO REALLY DELIGHTED
TO SEE THIS HAPPEN. SHE RECEIVED
THIS AWARD A FEW WEEKS AGO
PERHAPS NOW BUT ALSO GOING TO
TELL YOU THERE'S A FEW OTHER HHS
AWARDS THAT I WANT TO TELL YOU
ABOUT. AND THIS IS HOT OFF THE
PRESSES BECAUSE THE PHOTO THAT
IS IN HERE ON THE RIGHT HAND
SIDE WAS TAKEN I THINK LITERALLY
AN HOUR AGO. SO THESE AWARDS ARE
BEING GIVEN TODA TODAY. THIS ISE
SECRETARY'S AWARE FOR
DISTINGUISHED SERVICE. THE
HIGHEST AWARD GRANTED BY THE
DEPARTMENT. AND THIS IS FOR THE
CREATION AND ESTABLISHMENT OF
THE NATIONAL -- NCATS NATIONAL
COVID COHORT COLLABORATIVE OR N
3C AS PEOPLE KNOW. SAM MICHAEL
IS IN THE CENTER NEXT TO
SECRETARY BECERRA AND THE TEAM
HERE ON THE LEFT-HAND SIDE ARE
THE ONES WHO HAVE BEEN REALLY
THE LEADERSHIP OF THIS PROGRAM
THAT SAM IS ABLE TO REPRESENT US
AND PICK UP THIS AWARDS FOR THIS
WORK. SO WE ARE OVER THE MOON
EXCITED THAT THE SECRETARY OF
HHS IS REALLY HAS RECOGNIZED THE
WORK N 3C HAS DONE IN THIS SPACE
AND WE HOPE TO CONTINUE THIS
WORK AS WELL AS IT HAS BEEN
PIONEERING IN THE WORK THAT WE
HAVE BEEN DOING IN CLINICAL
INFORMATICS. I WANT TO THANK THE
TEAM HERE THAT HAS BEEN
RECOGNIZED THE LEADERS OF THIS
PROGRAM. AGAIN, REFLECTING THE
BROAD EXTRAMURAL COMMUNITY
INVOLVED IN THIS CTSA
ESPECIALLY. AND MANY OTHER
PEOPLE WHO REALLY MADE THIS
HAPPEN ALONG WITH COLLEAGUES AT
NIH, PARTNERS IN THIS AS WELL.
THAT IS NOT IT. TURNS OUT THAT
THE SECRETARY'S AWARD FORMER
TORRIOUS SERVICE, THE SECOND
HIGHEST AWARD GRANTED BY THE
DEPARTMENT, ALSO RECOGNIZED THE
NCATS OPEN DATA PORTAL TEAM, SO
THIS IS A GROUP LED BY MATT HALL
AND HE'S THE ONE WHO IS
ACCEPTING THE AWARD ABOUT AN
HOUR AGO DOWNTOWN RECEIVING THIS
AWARD AND SENDING ME THE PHOTOS
WE HAVE LEADERSHIP TEAM SHOWN
HERE REFLECTIVE OF COURSE ACROSS
A VARIETY OF GROUPS, THIS IS A
PUBLIC PRIVATE PARTNERSHIP FOR
EXAMPLE THAT REALLY MADE THIS
HAPPEN. BLUE I WAS EXCITED TO
SEE AGAIN NCATS RECOGNIZED FOR
THE KEY WORK WE DO BUT IT IS
ALWAYS ABOUT THE PEOPLE
UNDERLYING THAT MAKE IT HAPPEN I
WANTED TO MAKE SURE I WAS ABLE
TO RECOGNIZE THEM HERE TODAY.
ONE OTHER AWARD IS SORT OF
UNUSUAL AND FELT IT WORTHY TO
HONOR THIS GROUP, DR.
(INAUDIBLE) RECEIVED A HONORARY
AWARD FROM THE UNIVERSITY OF
SIDNEY STRAYIA. THE HIGHEST
AWARD BEST STOWED UPON GRADUATES
FROM UNIVERSITY OF SIDNEY. IT
RECOGNIZES HIS WORK ON
SUPPORTING LNI RELATIONS FOR THE
UNIVERSITY AS WELL AS
OUTSTANDING SCIENTIFIC
ACCOMPLISHMENTS. SINCE THEN. SO
RECOGNIZING FROM AFAR FROM DOWN
UNDER AND I'M REALLY EXCITED TO
SEE THIS RECOGNITION HAPPEN AS
WELL FOR NCATS STAFF SO
CONGRATULATIONS TO ALL OF THOSE
FOLKS WHO REALLY BEEN AT THE
FOREFRONT THE LAST COUPLE OF
YEARS LEADING IN THIS SPACE, IN
THESE SPACES. SO IT IS SORT OF
AS WE COME BACK OUT OF THE
PANDEMIC, THIS IS WHERE YOU KNOW
OF THE FIRST TIMES WE HAD TO
HOLD NIH RESEARCH FESTIVAL THAT
IS SOMETHING THAT IS FOR
INTRAMURAL RESEARCH PROGRAM. SO
I WANTED TO JUST DEPICT A
VARIETY OF OUR TRAINEES AND POST
DOCS WHO WERE PRESENTING POSTERS
AT THE RESEARCH FESTIVAL. M CATS
HAS AMAZING PRESENCE THERE. WE
WERE REPRESENTED 3.5 FOLD MORE
THAN OTHER ICs SO I'M EXCITED
TO SEE THE SCIENCE CONTINUE AND
ADVANCEMENT THEY BRING TO THIS
RESEARCH FESTIVAL, IT IS ALWAYS
IMPORTANT, THIS IS PART OF
TRAINING, YOU GO AND PRESENT
POSTERS, TALK TO PEOPLE TELL
THEM ABOUT THE WORK YOU DO. THIS
IS HELPING US DEVELOP TRAINEES
AND FUTURE LEADERS FOR
TRANSLATIONAL SCIENCE SO EXCITED
TO HAVE THIS BATCH IN THE NIH
FOLD THAT WE ARE ABLE TO
PARTICIPATE IN IN AGAIN AND I
LOOK FORWARD TO FUTURE NIH
RESEARCH FESTIVALS AS WELL. ALL
RIGHT. NOW I WANT TO MENTION A
FINISHTIVES THAT NCATS HAS BEEN
PARTICULARLY INVOLVED IN OVER
THE LAST FEW MONTHS SINCE WE MET
LAST. WE HAVE BEEN OF COURSE
WORKING THE HELPING END
ADDICTIONS LONG TERM SPACE AND
THIS IS THE HEAL SPACE. SO NO
STRANGER TO THIS PROGRAM BUT
THERE IS A NEW INITIATIVE WE ARE
EXCITED ABOUT THE ROLE IS UNIQUE
AND THIS IS A PARTNERSHIP WITH
HELP FROM ANNA RAMSEY EWING AND
HER OFFICE AND STACY FLETCHER.
THIS IS THE NCREW PROGRAM, A
PRESTIGIOUS PROGRAM WITHIN THE
HEAL COMMUNITY TO HELP US
CONSIDER HOW TO BRING IN AND
RESPOND TO THE NEEDS WITHIN THE
TRIBAL COMMUNITIES. SO THIS
EFFORT IS TO SUPPORT TRIBES
NATIVE AMERICANS SERVING
ORGANIZATIONS TO CONDUCT THE
LOCAL RESEARCH TO ADDRESS
OVERDOSE, SUBSTANCE USE PAIN AND
INTERSECTIONS WITH THOSE ALONG
WITH OTHER HEALTH AND WELLNESS
FACTORS SO THIS N CREW
INITIATIVE IS OUT. AND WE ARE
PARTNERING ON THIS WITH THE
NATIONAL INSTITUTE ON DRUG ABUSE
WHO WILL HAVE OVERALL PROGRAM
OVERSIGHT. BUT BECAUSE OF THE
NATURE OF THIS AWARDS, THE
PROGRAMMATIC OVERSIGHT GOES HAND
IN HAND WITH THE SORT OF OTHER
TRANSACTION AWARDS AND
ADMINISTRATIVE AND REVIEW
OVERSIGHT. THAT IS WHAT WE
BRINGING TO THE TABLE. WE ARE
VERY EXCITED ABOUT THIS BECAUSE
THIS WILL ALSO I THINK
STRENGTHEN OUR OWN ABILITIES TO
UNDERSTAND WHAT IS NEEDED FOR
THESE TYPES OF COMMUNITIES AND
HOW TO EXPAND TO OTHER
COMMUNITIES AS WELL THAT MAY NOT
NECESSARILY BE AS TRADITIONAL
KINDS OF NIH AWARDEES. SO THIS
MIGHT HELP EXPAND OUR REPERTOIRE
IN THAT WAY. SO WE ARE EXCITED
ABOUT IT FOR THIS REASON. NOT
ONLY THE SCIENTIFIC WORK AT N
CATS BUT OPERATIONAL WORK THAT
WE DO TOO. THAT HELP US LEAD AN
INNOVATE IN THIS SPACE SO I WANT
TO LET YOU KNOW WE ARE INVOLVED
IN THIS, IF YOU WANT TO KNOW
MORE ABOUT THE PROGRAM I HAVE
THE QR CODE THERE. RECENTLY I
THINK THIS CAME OUT THE LATEST
DIRECTORS BLOG FROM DR. TABAK,
HE TALKED ABOUT THE TARGETED
CHALLENGE THAT WE HAVE, THE
TARGETED GENOME EDITOR DELIVERY.
THIS IS A CHALLENGE WE HAVE LIEU
THE COMMON FUND SOMATIC CELL
GENE EDITING PROGRAM THAT NCATS
IS ONE OF THE LEADERS OF ALONG
WITH NINDS AND WALTER KOROSHETZ,
PJ BROOKS IS LEAD AT NCATS WHO
IS REALLY HELPED DEVELOP THIS
CHALLENGE BUT THIS IS TO HELP
GET TARGETED DELIVERIES TO
UNUSUAL PLACES IN THE BODY AND
UNDERSTAND HOW IT CAN INNOVATE
IN THIS SPACE TO MAKE GENE
EDITING A VARIETY FOR DIFFERENT
KINDS OF DISEASES. IF YOU ARE
INTERESTED IN THIS THE QR CODE
IS THERE AND CHALLENGE IS NOW
OPEN. LASTLY ANOTHER NEW
INITIATIVE THAT I WANT TO
PRESENT IS ONE CALLED
COMPLIMENTARY. LUCKILY THIS IS
NOT A FULL ACRONYM ONLY THE ARIE
IS THE ACRONYM BUT COMPLIMENTARY
IS THE FULL NAME OF THE PROGRAM
AND THIS IS ABOUT COMPLIMENTING
ANIMAL RESEARCH AND
EXPERIMENTATION. THIS BUILDS
FROM LAST DECADE OF WORK THAT
DAN TAGLEY AND HIS TEAM LED IN
THE MICROPHYSIOLOGICAL SYSTEMS
SPACE TO THINK HOW TO BRING THAT
-- THOSE TISSUE CHIP KINDS OF
PROGRAMS THE 3-D PROGRAMS TO
BEAR ON EARLY EXPANDING AND
INNOVATING IN THE RESEARCH SPACE
AND PERHAPS ADDRESSING HOW WE
CAN COMPLIMENT ANIMAL RESEARCH
AND EXPERIMENTATION. NOT REDUCE,
NOT REPLACE, BUT -- NOT TO
REPLACE BUT CERTAINLY REDUCE AND
HELP US UNDERSTAND WHERE AND HOW
IT IS GOING TO BE APPROPRIATE TO
USE THESE SORTS OF NEW MODELS.
BUT IT WON'T BE ALL THE TIME
THAT WE ARE GOING TO BE ABLE TO
JUST USE THESE MODELS IN PLACE
OF ANIMALS. THEY ARE ANIMAL
MODELS ARE STILL INCREDIBLY
IMPORTANT AND WE NEED TO MAKE
SURE WE KNOW WHEN AND HOW TO USE
THE DIFFERENT MODELS MOST
APPROPRIATE FOR THE QUESTI QUESO
THE PURPOSE OF THE PROGRAM IS
CATALYZE DEVELOPMENT AND
STANDARDIZATION VALIDATION OF
THESE KIND OF APPROACHES. SO
THESE ARE SPECIFICALLY HUMAN
BASED APPROACH TO DEVELOP THOSE
NEW APPROACH METHODOLOGIES THAT
WILL HOPEFULLY TRANSFORM HOW WE
THINK ABOUT TRANSLATIONAL AND
CLINICAL SCIENCE. IN VITRO
INNOVATIONS IN SILICO INNOVATION
AND THE (INAUDIBLE) INNOVATIONS
SO USING THE TECHNOLOGIES WE
HEARD ABOUT AND BRINGING TO BARE
ON VARIETY OF DISEASES SO
BUILDING BETTER MODELS, GETTING
WAYS IN WHICH WE CAN PROVIDE
INITIATIVES TO GET INSIGHT INTO
SPECIFIC BIOLOGICAL PROCESSES.
HOW TO VALIDATE THESE MODELS
EFFECTIVELY AND THINK ABOUT WHEN
AND HOW TO USE THEM FOR
REGULATORY USE OR WHEN AND HOW
TO USE THEM FOR EXPERIMENTAL
USE. THEN COMPLIMENTING THESE
DIFFERENT MAMMALIAN MODELS IS
SOMETHING THAT WILL BE
IMPORTANT. GENORA I WILL
UNDERSTANDING HOW WE CAN MAKE
THIS MORE EFFICIENT AND
EFFECTIVE FOR DRIVING THE
PROCESS OF TRANSLATIONAL SCIENCE
FASTER AND BETTER MORE
EFFECTIVELY. THERE HAVE BEEN A
VARIETY OF PLANNING EFFORTS
UNDERWAY FOR THIS PROGRAM BUT
ONE THAT IS CURRENTLY OUT IS
THAT WE ARE DOING IT A LISTENING
SESSION FOR THE COMMUNITY. SO
THIS IS TO JUST UNDERSTAND WHAT
THE NEEDS ARE IN THE COMMUNITY
AND UNDERSTAND WHAT KINDS OF
INSIGHTS PEOPLE ARE LOOK FOR,
FOR HOW THE COMPLIMENTARY
PROGRAM CAN REALLY PROVIDE
OPPORTUNITIES AND START TO
ADDRESS ROAD BLOCKS THAT ARE
CURRENTLY ONGOING IN THE NOVEL
APPROACH METHODOLOGIES
DEVELOPMENT. SO THE THREE ROUND
TABLES OR LISTENING SESSIONS
THAT WILL BE CONDUCTED ARE SHOWN
HERE. ALL IN OCTOBER. THERE ARE
SECTIONED OUT BY DIFFERENT
SECTORS THAT WILL BE LISTENING
TO AT THOSE TIMES. IF YOU ARE
INTERESTED IN JOINING ANY OF
THOSE TAKE A NOTE OF THE QR CODE
HERE AND YOU CAN REGISTER FOR
THOSE ACTIVITIES. THEN AFTER
THAT WILL BE THINKING ABOUT A
BROADER LANDSCAPE ANALYSIS, AND
THIS IS GOING TO BE IN
CONJUNCTION WITH THE ACTIVITIES
THAT ARE ACTUALLY ONGOING IN THE
ADVISORY COMMITTEE TO THE
DIRECTOR. THAT -- WHO HAS A
WORKING GROUP DEDICATED TO NOVEL
APPROACH METHODOLOGIES. SO THEY
HAVE BEEN DOING WORK OVER THE
LAST SIX MONTHS, NINE MONTHS, I
GUESS SEPTEMBER, THE LAST NINE
MONTHS AND THEY HAVE A FEW MORE
MONTHS TO GO THEN PROVIDE
RECOMMENDATIONS TO THE NIH AT
THE END OF THE YEAR. BASED ON
THOSE RECOMMENDATIONS WE WILL
INTEGRATE THAT INTO THE -- WHAT
WE ARE DEVELOPING FOR
COMPLIMENTARY, SO WE CAN REALLY
GENERATE A VERY WELL INFORMED
AND ROBUST PROGRAM FOR THE
COMMON FUND PROGRAM TO PURSUE
THE NEXT SEVERAL YEARS. STAY
TUNED. AND WE ARE EXCITED THAT
THE WORK THAT WE HAVE BEEN DOING
HAS BEEN ABLE TO FOLD INTO
SOMETHING OF THIS NATURE. WE ARE
GRATIFIED TO SEE THAT WORK. ONE
LAST THING TO HIGHLIGHT THAT HAS
ACTUALLY COME TO A CULMINATION
OVER MORE THAN FIVE YEARS OF
WORK INCLUDING DURING THE
PANDEMIC, THIS WAS STARTED WITH
MY PREDECESSOR CHRIS AUSTIN AND
VARIETY OF OTHER FOLKS AT
NATIONAL ACADEMIES OF MEDICINE.
WHEN I JOIN AD FEW YEARS AGO
DURING THE PANDEMIC I AM PART OF
DRUG DISCOVERY DEVELOPMENT
TRANSLATION FOR THE NATIONAL
ACADEMIES SO PART OF MY
AGREEMENT WAS TO HELP WORK
THROUGH AND FINALIZE THIS
FRAMEWORK FOR ASSESSING CLINICAL
TRIALS SITE READINESS. THIS IS
SEEN AS A REAL NEED WITHIN THE
COMMUNITY TO HELP PROVIDE
GUIDANCE AND RESOURCES FOR
SIGHTS OUT THERE DOING CLINICAL
TRIALS. HOW WE CAN START TO
BRING THESE TOGETHER TO MAKE
THEM MORE STREAMLINED FOR A
VARIETY OF REASONS. THERE ARE
FOUR PIECES THAT WILL BE
PUBLISHED ON THIS COMPENDIUM
GROUP OF PAPERS BEING PUT
FORWARD. THE MAIN IS FRAMEWORK
FOR ASSESSING CLINICAL TRIAL
SITE READINESS AT THE TOP BUT
THERE ARE THREE PARALLEL PAPERS
OR PERSPECTIVES. ONE IS ACTUALLY
CONSIDERATIONS FROM THE CTSA
PROGRAM. AND SO THERE ARE IS --
I WILL TALK MORE ABOUT THAT.
THEN THERE WILL BE ONE -- THERE
ARE TWO IN PRESS, ONE ON THE
DIVERSITY EQUITY INCLUSION
ACCESS NEEDS AND THINKING ABOUT
CLINICAL TRIALS SITE READINESS
SPECIFICALLY. AND THEN ALSO ON
DECENTRALIZED TRIALS SO TWO
OTHER PERSPECTIVES COMING OUT
BUT CURRENTLY IN PRESS SO I
WASN'T ABLE TO SHOW YOU THAT
TODAY. SO A LITTLE BIT MORE
WHAT THIS WORK WAS ALL ABOUT WAS
TRYING TO UNDERSTAND WHAT THIS
FRAMEWORK SHOULD LOOK LIKE. SO
THERE WERE SOME FOUNDATIONAL
COMMON PRINCIPLES IDENTIFIED AS
CORE TO SITE READINESS FOR
CLINICAL TRIALS. A CULTURE OF
QUALITY AND THINKING HOW TO BE
IN THIS CONTINUOUS NEED OF
INCREMENTAL IMPROVEMENT OVER THE
COURSE OF DOING CLINICAL TRIALS
FOR ANY SITE OUT THERE. TO BE
THINKING HOW PERSON CENTEREDNESS
SHOULD BE AT THE CENTER OF
CLINICAL TRIAL SITE READINESS,
TO BE RESPECTING FOR RESPECTING
OF HUMAN RIGHTS AND WELFARE FOR
TRIAL PARTICIPANTS AND MAKING
SURE THAT THAT IS WELL INFORMED
ENGAGED. AND CLINICAL RESEARCH
LITERACY, THINKING ABOUT HOW TO
ENGAGE AND INFORM STAKEHOLDERS
THAT ABOUT THE SITE READINESS
PROGRAMS AND TO ELEVATE
CONFIDENCE IN THE CLINICAL
TRIALS CONDUCTED. SO THAT IS
THE WRAPPER AROUND THEN SIX
DIFFERENT DOMAINS OF CLINICAL
TRIALS SITE READINESS THAT THIS
BODY HAS COME TOGETHER ON.
THROUGH A VARIETY OF LOOKING AT
OVER 200 OF THESE SORTS OF
FACTORS THAT GO INTO CLINICAL
TRIAL SITE READINESS, LOOKING AT
WHAT THE IS CRUX OF THOSE WHAT
IS COMMON AMONG THEM. GOING
THROUGH A SEMIDELPHI METHOD TO
WHITTLE THIS DOWN TO THE VERY
IMPORTANT PIECES HERE. SO
RESEARCH TEAMS INFRASTRUCTURE
STUDY MANAGEMENT, DATA
COLLECTION MANAGEMENT, QUALITY
OVER SIGHT, ETHICS AND SAFETY
ARE THE SIX DOMAINS OF FRAMEWORK
FOR ASSESSING CLINICAL TRIAL
SIDE READINESS. IN THE PAPER IT
OUTLINES VERY SPECIFIC KEY
OBJECTIVES UNDER EACH OF THESE
SIX DOMAINS THAT WE PROVIDE TO
ANY CLINICAL TRIAL SITE OR ANY
SITE LOOKING TO BE CLINICAL
TRIAL SITE READY. SO THEY CAN
PERFORM THEIR OWN
SELF-ASSESSMENT IF YOU WILL.
WHERE DO THEY STAND? WHERE ARE
THEY IN THE STATUS OF THESE KEY
DOMAINS AND HOW DO THEY NEED TO
ELEVATE THOSE OR MAKE SURE THEY
ARE CONTINUING TO MAINTAIN THE
HIGH LEVELS THEY ARE AT
CURRENTLY. THIS IS THE IDEA, FOR
EXISTING SITES, IT IS ALSO FOR
NEW SITES TO HELP COMING ON
BOARD PART OF CLINICAL TRIAL
INFRASTRUCTURE I WANT TO
HIGHLIGHT FROM THE CTSA PROGRAM,
SPECIFIC COMMENTS MADE IN TERMS
OF DEVELOPING STANDARDIZED
OPERATING PROCEDURES WITHIN
INSTITUTIONS AN COMMUNICATING
WITH THE CLINICAL TRIAL SITES
WITHIN INSTITUTIONS AT THE
ACADEMIC MEDICAL ORGANIZATIONS,
HAVING LEADERSHIP UNDERSTAND
WHAT THE NEEDS ARE TO MAKE SURE
THOSE ARE MAINTAINED.
DEVELOPING STANDARDIZED THEM
PLACE IS IMPORTANT AND AS WELL
AS IMPLEMENTATION PLANS FOR
TRANSITIONING TO NEW PROCEDURES.
SO IF AND WHEN OR IF SAY
PANDEMIC WERE TO COME ACROSS OUR
WAY AGAIN, PERHAPS THESE ARE
SOPs THAT CAN BE PULLED OFF
THE SHELF, DUSTED OFF A BIT, AND
REFINED TO MEET THE NEEDS OF
WHAT IS HAPPENING SO THIS WOULD
BE CERTAINLY THE IDEA BUT YOU
DON'T NEED A PANDEMIC FOR ANY
NEW CLINICAL TRIAL. THESE ARE
PLANS YOU CAN CONTINUE TO
MAINTAIN. REALLY THINKING ABOUT
THESE HOW TO HARMON RECOGNIZE
DOCUMENTATION ACROSS THE SITES O
SO THERE'S INTEROPERABILITY
ACROSS THE INFRASTRUCTURE
GLOBALLY OF THE CLINICAL TRIAL
ENTERPRISE. THESE ARE KEY
FEATURES THE CTSA PROGRAM CAME
OUT WITH, I WANT TO HIGHLIGHT
THE QR CODE FOR THIS
PUBLICATION. WHY IMPORTANT?
THERE'S TWO MORE PUBLICATIONS
COMING TO STAY TUNED FOR THOSE.
BUT I THINK THIS IS IMPORTANT, I
REALLY WANT TO RELATE TO THIS
GROUP SPECIFICALLY, HAVING THIS
PER SHARED UNDERSTANDING OF SITE
READINESS WILL HELP US BE ABLE
TO HAVE A LOT MORE CONFIDENCE
NOT JUST WITHIN OURSELVES BUT
STUDY SPONSORS AND ALSO THE
CLINICAL TRIAL PARTICIPANTS WHO
RELY ON US TO CONDUCT THESE
TRIALS AND PROVIDE EVIDENCE FOR
SUPPORT OR NOT FOR WHAT WE
TRYING TO ADDRE ADDRESS. WE HOPE
FRAMEWORK LOWERS BARRIERS FOR
ENTRY FOR SITES TO DO SELF
ASSESSMENT, WHAT THEY NEED TO DO
FOR CLINICAL TRIAL SITE
READINESS. MAINTAINS CONFIDENCE,
AND ALSO HELP INCREASE DIVERSITY
AND INCREASE THE
GENERALIZABILITY OF CLINICAL
TRIALS THAT WE SUPPORT. SO I
HOPE THESE TYPES OF OR THIS
FRAMEWORK AND THE TYPES OF IDEAS
THAT ARE GENERATED HERE ARE
REALLY EMBRACED BY THE COMMUNITY
AND MAYBE WE CAN EVEN TAKE A
STEP FURTHER AND PROVIDE BEST
PRACTICES AS WE START TO LEARN
MORE AND KICK THE TIRES HOW
IMPORTANT EACH OF THESE ARE. SO
I HOPE THAT THIS WILL BE HELPFUL
OF VARIETY OF YOUR PARTNERS AND
COMMUNITIES. SO I WANT TO TAKE A
FEW MINUTES AND END ON THE
STRATEGIC PLAN PROCESS. I WANT
TO HAVE A FEW SLIDES TO CATCH
YOU UP ON WHAT WE HAVE BEEN
DOING SINCE WE MET. WE ARE NOW
AT THE POINT WE HAVE WRITTEN RFI
AND THE RFI IS OUT ON THE
STREET. AND THAT IS A QR CODE
FOR THE RFI, YOU CAN TAKE A
PICTURE BUT I HAVE MORE QR CODES
LATER ON THE RFI SO IF MISSED
NOW YOU CAN GET IT LATER BUT WE
ARE EXCITED TO BE AT THIS STAGE.
THE RFI IS INTERIM. AND IT IS
MEANT TO SAY GOSH, WE HAVE HAD
NEARLY 50 POUNDS TABLES TO HEAR
INPUT AND FEEDBACK ON WHAT NCATS
IS DOING WHERE WE WANT TO GO,
OUR HIGH LEVEL MORE TREATMENTS
FOR ALL PEOPLE MORE QUICKLY
AUDACIOUS GOALS, HOW WE CAN
ALIGN THOSE WITH HOW WE CAN MOVE
NEXT. WE ENGAGED 1150
INDIVIDUALS AND HAD OVER 1700
UNIQUE COMMENTS DURING THIS
PROCESS. A ARE THE OF DATA; WE
DON'T HAVE NATURAL LANGUAGE
PROCESSING WELL HONED FOR THIS
PROCESS BUT BEST WE COULD IN
EMERITUS TEMPLE O'CONNER AND HER
BRAIN AND TEAM PULLING THIS
TOGETHER TO MAKE IT HAPPEN. WHAT
WE HAD A VARIETY OF LISTENING
SESSIONS WITH A VARIETY OF
CONSTITUENT SAYS AND I WANT TO
GIVE YOU A SENSE AND I AT A
GLANCE SNAP SHOT WHAT WE HEARD.
SO FOR EACH OF THESE DIFFERENT
GROUPS OR STAKEHOLDERS WE HAD,
WE HAD A VARIETY OF TOPICS. YOU
WILL SEE THOSE TOPICS ON THE
RIGHT HAND SIDE WHEN YOU HEAR
1790 UNIQUE COMMENTS WE WERE
ABLE TO BOIL THEM INTO A ROLE UP
INTO HIGH TOPIC CONSIDERATIONS.
SO YOU CAN SEE HOW THOSE
COMMENTS PLAYED OUT WITH THE
DIFFERENT LISTENING SESSIONS. T
SO I WANTED TO JUST GIVE YOU A
SENSE OF VARIOUS AREAS
COLLABORATION PART SHIPS, DATA
SCIENCE, COMMUNITY ENGAGEMENT,
WORK FORCE DEVELOPMENT, DEIA,
RARE DISEASE, AND HOW WE SET
PRIORITIES ALL TOPICS RELAY AND
COMMON THEMES WE PULLED OUT OF
THAT. SO AS WE GO FORWARD, THIS
IS A SPAGHETTI DIAGRAM OF
INPUTS, SO YOU CAN SEE THE
PRIORITIZATION START TO SHAKE
OUT BASED ON THE DIFFERENT
COMMUNITY NEEDS SO WE CAN START
TO TAILOR THIS TO THE OTHER
PROGRAMS NCATS SUPPORTS BUT WE
WANT TO MAKE SURE THAT WE HAVE
BEEN HEARD THE COMMUNITY IN THE
RIGHT WAY SO ESSENTIALLY WE HAVE
COME UP WITH THIS IDEA OF THE
DRAFT FRAMEWORK, MORE TREATMENTS
MORE PEOPLE MORE QUICKLY BUT IT
TRANSLATES TO THE THREE PURPLE
SECTION, APPLYING APPROACHES TO
FOSTER THE IDENTIFICATION AND
DEVELOPMENT OF ACCESS TO MORE
TREATMENT. ENABLING PEOPLE TO
CONTRIBUTE BENEFIT FROM
TRANSLATIONAL SCIENCE.
ACCELERATING TRANSLATION BY BOTH
ADDRESSING THE SCIENTIFIC AND
OPERATIONAL CHALLENGES. AND OF
COURSE CROSS CUTTING ALL THOSE
ARE A VARIETY OF AREAS IN THOSE
OPERATIONS THAT CUT ACROSS THE
WORK THAT WE DO. AND HOW TO
ADVANCE TRANSLATIONAL SCIENCE.
SO THOSE ARE START TO PUT MEAT
ON THE BONES WHEN WE GET RFI
BACK FROM THE COMMUNITY. SO HERE
AGAIN IS THE PHOTO OF THE RFI. I
WOULD ENCOURAGE YO YOU TO PLACE
TAKE A LOOK AT THE REQUEST FOR
INFORMATION. SEND TO YOUR
FRIENDS AND COLLEAGUES, WE WANT
YOUR FEED BECOME AND PAY
ATTENTION TO SOME OF THE
QUESTIONS WE ASK THERE. BUT ALSO
DIDN'T WE HEAR YOU RIGHT? WHAT
FROM KEY THINGS WE MISS? WE
DON'T WANT TO REHASH THE WHOLE
IDEA BUT WE WANT TO MAKE SURE WE
DIDN'T MISS ANYTHING MAJOR AND
WE ALSO WANT TO MAKE SURE WE
HEAR YOU ON HOW WE PRIORITIZE
THESE ASPECTS AND HOW WE THINK
ABOUT METRASIZING AS WELL SO
THESE ARE THE THINGS WE ARE
ASKING DURING THIS RFI TIME
FRAME. SO CLOSES NOVEMBER 1 AND
WE LOOK FORWARD TO REVIEWING
THAT FEEDBACK AND COMING UP WITH
THEN OUR FULL PLAN AFTER THAT.
AFTER THAT OUR TEAM WILL TAKE IT
FROM THERE AND TRY TO SIT DOWN
AND DEVELOP IT. IT IS A
STRATEGIC PLAN, IT IS NOT A
TACTICAL PLAN BUT WE HOPE THE
STRATEGIES THAT WE OUTLINE WILL
PROVIDE A VERY CLEAR OBJECTIVES
THAT WE ARE LOOKING FORWARD TO
MAKE PROGRESS. WITH THAT, I
SUMMARIZE HERE, WE HAD AGAIN A
VARIETY OF IMPACTFUL ADVANCES
REFLECTED A LOT BY THE HONORS
AND RECOGNITION WE RECEIVED, THE
WORK WE ARE CONTINUING TO DO,
AND DECENTRALIZED TRIALS, AND
THE SITE READINESS FRAMEWORK AND
THE NEW COMPLIMENTARY PROGRAM AS
WELL. OUR RFI IS OUTS, WE ARE ON
TRACK FOR THE QUARTER 1 RELEASE
IN 2024. WITH THAT WE WILL STOP
THERE AND SEE IF THERE'S
QUESTIONS BEFORE WE MOVE ON TO
THE OTHER UPDATES WE WILL BE
DOING LATER TODAY FROM MIKE
KURILLA AND DIVISION OF CLINICAL
INNOVATIONS AND CHRISTINE
COLVIS, OFFICE OF DRUG
DEVELOPMENT PARTNERSHIP PROGRAM
I WILL STOP THERE. ANY
QUESTIONS?
>> I HAVE A QUESTION. JUST
BECAUSE I'M NOT QUITE FAMILIAR.
YOU MENTIONED IN THE CLINICAL
TRIALS READINESS FRAMEWORK
RESEARCH TEAMS AND YOU REFERRED
LATER ON ABOUT TRYING TO EXPAND
AND DIVERSIFY THEM. WHAT DOES
THE WORK FORCE LOOK LIKE RELATES
TO CLINICAL TRIALS RESEARCH
TEAMS AND TRIAL LIST, IS THERE A
SHORTAGE, WHAT DOES THE
DIVERSITY LOOK LIKE?
>> GREAT QUESTION. THAT SPECIFIC
QUESTION NATIONAL ACADEMIES,
WHAT THEY ARE DOING TOO, WE NEED
MORE THOUGHT AROUND WHAT THAT
WORK FORCE LOOKS LIKE. IT IS NOT
TO SAY THAT THAT WE DON'T
RECOGNIZE VARIOUS DIVERSITIES
NEEDING BIOSTATISTICIANS YOU
NEED CLINICIANS, YOU NEED PEOPLE
WHO CAN HELP PATIENTS NAVIGATE
THE PROCESS. PHARMACISTS. YOU
NEED ALL THE PEOPLE AT TABLE TO
DEVELOP CLINICAL TRIAL AND
PROTOCOLS TO MOVE THEM FORWARD.
SO OUTLEUPPS WHAT THOSE ARE AS
CAREER DEVELOPMENT PROGRAMS AND
ENGAGING AND MAKING
RECOMMENDATIONS TO INSTITUTIONS
PERHAPS TO HOW TO BUILD THEIR
PROGRAMS TO SUPPORT MAINTAIN,
THAT IS ACTUALLY A -- ANOTHER
NATIONAL ACADEMIES MEETING THAT
WILL BE HAPPENING LATER THIS
MONTH. I THINK MIKE KURILLA WILL
BE THE ONE ATTENDING. MIGHT BE
SOMEBODY ELSE. BUT WE HAVE A
ROLE IN THAT TOO, UNFORTUNATELY
I HAD A CONFLICT SO COULDN'T
MAKE IT BUT WE ARE EXCITED TO BE
PART OF THAT TOO BECAUSE IT
HELPS IDENTIFY HOW WE CAN HELP
THE COMMUNITIES PREPARE FOR K
SCHOLARS T SCHOLARS ET CETERA
AND HELPING DEVELOP THE NEEDS
AND WHAT THE CLINICAL TRIALS OF
THE FUTURE LOOKS LIKE. THE IDEA
OF CONSTANT IMPROVEMENT AND
ENGAGEMENT OF CLINICAL TRIALS IS
IMPORTANT FOR THIS VERY IDEA. AS
WE THINK ABOUT THE TYPICAL
CLINICAL TRIALS USUALLY HAVE
BEEN IN METHODS DEVELOPMENT OR
REGISTRATIONAL CLINICAL TRIALS
IN TERMS OF SMALL MOLECULE
MODALITIES. TESTING THEM IN THE
CLINICAL TRIAL SPACE. WITH NEW
MODALITIES THAT ARE COMING OUT
MORE AND MORE, WE ARE HEARING
ABOUT GENE THERAPIES. ANTISENSE
OLIGONUCLEOTIDES, GENE EDITING
THERAPIES. WE NEED TO BE
PREPARED FOR THE KINDS OF
CLINICAL TRIALS AND RESEARCH
NEEDED TO SUPPORT THOSE
ACTIVITIES. SO THAT CONSTANT
IMPROVEMENT AND ALWAYS CONSTANT
UNDERSTANDING WHAT THE WORK
FORCE NEEDS ARE ALONG THE WAY BE
MORE EFFICIENT TO MAINTAIN THE
READINESS ASPECTS WE ARE TRYING
TO GO FOR. IT IS ITERATIVE BUT
DEFINITELY PART OF THE
DISCUSSION. MATTHIAS.
>> THANKS A LOT FOR THE UPDATE
CERTAINLY CONGRATULATIONS TO
NCATS STAFF. I THINK FUTURE ON
CLINICAL TRIAL READINESS IS WE
ARE SEEING PHILOSOPHY OF
CLINICAL TRIALS ARE DONE
INCREMENTALLY CHANGE, HAS CHANGE
POSITIVE FOR THE WORK FORC FORCO
GOOD NEWS RECEIVE RESEVERAL
ACTIVITY WHICH COVERS MANY
DOMAINS AND ALSO MORE LATER THIS
AFTERNOON WE WILL HEAR
INNOVATIVE SOLUTIONS. IT IS
CLEAR THAT ALL THOSE TOOLS
PARTICULARLY MINORITIES,
PARTICULARLY BRINGING STUDIES TO
PEOPLE AND MORE PEOPLE TO
STUDIES, WITH RESPECT TO THEM,
SO OTHER COMMON DISEASES ARE
ASSAY TO BE DEVELOPED, EXPECTED
TO CTSA, AS (INDISCERNIBLE) BUT
ALSO AS PROVIDING ON TOPICS
(INAUDIBLE). WORK FORCE ISSUE IS
REAL, IT HAS SIGNIFICANTLY
ACCELERATED PANDEMICS IN OUR
FIELD OF KIDNEY RESEARCH,
SOMETIMES USE AWARD CRISIS THAT
WE REALLY HAVE NOT PARTICULARLY
STAFFED TO BRING OUR STUDIES AND
(INAUDIBLE) WHICH ARE FINALLY
AVAILABLE TO OUR CLIENTS. THIS
APPEARS TO LESSER EXTENT TO BE
THE CASE IN PARTS OF EUROPE,
JAPAN, THE INFRASTRUCTURE HAS
RECOVERED MORE QUICKLY. AND IS
FULLY (INAUDIBLE) COMPARE TO
WHAT WE HAVE SEEN HERE. ONE
FOCUS TO KEEP INCLUSIVE
PARTICULARLY IN TIMES OF
(INAUDIBLE) NOT ONLY FOCUS ON
THE M.D. Ph.D. LEVEL
SCIENTISTS BUT ALSO TO STAFF
SCIENTISTS. THOSE ARE THE ONES
WHO IN OUR EXPERIENCES RIGHT NOW
MANY ACTIVITIES SO FINDING WAYS
TO BUILD CARRIER (INAUDIBLE)
EXCITING INNOVATIVE AND WHO HAVE
SECURITY ATTACHED TO THEM,
SOMETHING I THINK NCATS MIGHT BE
ONE OF THE TREATMENTS.
>> THANK YOU VERY MUCH FOR THAT
COMMENT. YOU ARE EXACTLY RIGHT.
THAT SOMETHING WE HAVE TALKED
ABOUT THROUGH THE NATIONAL
ACADEMIES IS EXACTLY IDEA OF HOW
TO BUILD AND SUSTAIN THOSE
CAREER PATHS THAT ARE NOT
M.D.s AND Ph.D.s. HIGH
LEVEL DEGREES. THESE ARE
CRITICAL CAREER POSITIONS THAT
ENABLE A VERY ROBUST CLINICAL
TRIAL WORK FORCE. MAKING THOSE
ATTRACTIVE TO PEOPLE IS
SOMETHING THAT IS OF INTEREST
WITH THAT GROUP AND HAS BEEN
DISCUSSED. I AGREE WITH YOU AS
WELL, THIS IS PERHAPS AN AREA
WHERE NCATS CAN PLAY A ROLE
SUPPORTING SOME OF THAT
DEVELOPMENT. I APPRECIATE THAT
COMMENT FOR SURE. FOR THE
CLINICAL TRIAL SITE READINESS,
NOW THIS IS COMING OUT I HOPE
THAT WE WILL START TO HEAR HOW
IT IS HELPED INFORM OR SHAPE
WHAT PEOPLE ARE DOING ON BOOTS
ON THE GROUND HOW THEY ARE
THINKING ABOUT THAT SO WE CAN
START TO INSTANTIATE THEM MORE,
PERHAPS AS BEST PRACTICES AS I
MENTIONED. AND THOSE ARE AREAS
WE CAN MAKE SURE WE WILL PROVIDE
THAT ROBUST NATURE AROUND THE
CLINICAL TRIAL SO I COULDN'T
AGREE MORE, YOU MENTIONED THE
KIDNEY WORK YOU DO AND RARE
DISEASE SPACE, SO MUCH
INNOVATION IS NEED IN THAT SPACE
AND THIS WILL INFORM THAT WORK
NATIONALLY AND INTERNATIONALLY.
OTHER COMMENTS? QUESTIONS?
>> JUST A COMMENT ON THE LAST
COMMENT. I DO AGREE, THESE
POSITION PAPERS OR THESE
FRAMEWORK PAPERS WITH INPUT FROM
NCATS AND LEADERSHIP FROM NCATS,
THOSE TEND TO BECOME MATURITY
MODELS OVER TIME, THAT IS A GOOD
INVESTMENT. THANK YOU.
>> THANK YOU VERY MUCH. THIS WAS
NOT AN EASY TASK. THIS TOOK A
LOT OF TIME, A LOT OF DEBATE.
THAT JUST GOES TO THIS POINT
THAT WE ARE TRYING TO GET TO
ESSENCE, WHAT CAN WE PROVIDE
CHEWABLE, DIGESTIBLE, NOT SURE
OF THE WORLD BUT MEANINGFUL
IMPACTFUL THAT CAN BE USED AND
TAKEN UP SO WE SPENT TIME
THINKING ABOUT THAT, IT IS THE
COLLECTIVE OFFER PEOPLE WHO ARE
INVOLVED IN CLINICAL TRIALS BUT
THAT IS WHAT THE NATIONAL
ACADEMIES IS ABOUT, BRINGING
PEOPLE TO TABLE WITH THAT
EXPERTISE TO MAKE THAT HAPPEN.
SO I HOPE THIS, MY FINGERS ARE
CROSSED, IT WILL BE SOMEWHAT
THAT MATURITY MODEL YOU TALK
ABOUT, SEEMS LIKE THAT IS WHAT
THE CTSAs DO REALLY WELL IS
REALLY DEVELOP THOSE MATURITY
MODELS. SO THIS IS AN
OPPORTUNITY FOR US TO LEAD IN
THAT SPACE FOR SURE. I STAND UP
TRIAL READINESS INFRASTRUCTURE
FOR LUPUS. LUPUS RESEARCH
ALLIANCE I'M PART O. WE PROVIDED
MONEY AS PRIVATE FUNDER FOR
CLINICAL TRIAL READINESS AT 57
ACADEMIC MEDICAL CENTERS. THERE
IS A VOICE I WANT TO HE WILL
INVESTIGATE, CO-CHAIR OF
CLINICAL INVESTIGATORS NETWORK
IS THE CHAIR OF RHEUMATOLOGY AT
UCSF. SHE IS ABLE TO TAKE TIME
TO LEAD AND EMBRACE THE VALUE OF
CLINICAL TRIALS; OBVIOUS TO
EVERYONE IN THIS ROOM. SHE HAS
DECLARED A CRISIS IN CLINICAL
TRIALS FOR DOING RHEUMATOLOGY
STUDIES BECAUSE THE YOUNGER
GENERATION FEELS TOO MUCH
PRESSURE TO TRY TO BE ON TENURE
TRACK. SOMEHOW WE NEED THAT
CONVERSATION WITH THE NATIONAL
ACADEMIES TO ELEVATE ROLE OF
CLINICAL TRIALS BECAUSE WE ARE
HAVING PA TOUGH TIME GETTING THE
NEXT GENERATION THOSE THERE'S
FUNDING AVAILABLE THEY ARE NOT
SEEING IT AS GIVING THEM SKILLS,
WHAT TO DO HOW TO DO IT, IT IS
THE WRONG CHOICE IF THEY WANT TO
BE IN ACADEMIC MEDICINE. IT IS
DECLARED A CRISIS IN OUR
CLINICAL TRIAL NETWORK SO I'M
SHARING THAT AND MAYBE YOU WILL
HAVE AN OPPORTUNITY TO ELEVATE
THAT
>> ABSOLUTELY. THANK YOU FOR
SHARING THAT.LY KEEP YOU POSTED
ON THE CONVERSATIONS THAT HAPPEN
THAT HOPEFULLY CAN REFLECT THAT
INPUT. AND HIGHLIGHT AREAS WE
CAN PROVIDE GUIDANCE BACK. SO I
COULDN'T AGREE WITH YOU MORE,
BUILDS ON WHAT MATTHIAS
MENTIONED AS WELL, THIS IS AN
AREA IN NEED OF MANUFACTURE
CAREERS NECESSARY TO MAKE THIS
HAPPEN, SUPER IMPORTANT FOR
MAKING THIS SUCCESSFUL.
>> THIS IS PARTNERSHIPS WE
ESTABLISH OBVIOUSLY AS NIH MUST
BE TO TAKEN, 65% MEDICAL CENTERS
BANKRUPTCY ACADEMIC MEDICAL
CENTERS MEANING NO LEEWAY
AVAILABLE TO SUPPORT ACADEMIC
MISSION FOR CLINICAL (INAUDIBLE)
CUTTING ACROSS ALL MAJOR
(INAUDIBLE). THAT'S BEEN A
SIGNIFICANT RESOURCE IN THE PAST
IF YOU BUILD UP A FRAMEWORK YOU
HAVE TO INVEST FIRST TO GET YOUR
OPERATION OFF THE GROUND THEN IT
CAN HAPPEN BUT COMING UP
INNOVATIVE SOLUTIONS THE
INDUSTRY WHO OBVIOUSLY NEEDS US
CRITICALLY IN THAT PROCESS AS
WELL. MIGHT DECIDE TO FIND SOME
CREATIVE SOLUTION BEYOND
CRITICAL FUNDING SUPPORT.
>> EXCELLENT SUGGESTIONS ALL
AROUND. ANY OTHER THOUGHTS OR
COMMENTS?
>> DEBATING WHETHER THIS IS
NCATS MISSION BUT FEEL LIKE
FALLS WITHIN THE TWO MORE
PATIENTS MORE QUICKLY ELEMENTS
THINKING PUBLICATIONS AND WORK
YOU ARE DOING CTSA AND NATIONAL
ACADEMIES ALSO THINKING MORE
RECENT INNOVATIVE APPROVALS
WITHIN THE RARE DISEASE SPACE.
OFTEN TIMES THE CLINICAL TRIAL
SITES BECOME DELIVERY SITES FOR
THOSE INNOVATIVE THERAPIES.
THOSE ARE THE SITES THAT ARE
ABLE AS INNOVATIVE THERAPIES
BECAUSE THOSE ARE CRITICAL IN
PLACE GETTING TO THE PATIENT
MORE QUICKLY. SO CONSIDERED OR
HAVING THIS IN THE WORKS IS
TERRIFIC BUT TAKE THE SOPs AND
TRANSLATE INTO PROTOCOLS FOR
THERAPEUTIC DELIVERY, THAT CAN
BE DISSEMINATED BEYOND THIS
CLINICAL TRIALS SITES.
>> THAT IS A GREAT SUGGESTION.
WE PROBABLY COULD DO THAT NOW.
BUT I THINK FOR EXAMPLE, THE
WORK WE ARE DOING AT NCATS
THROUGH THE PEGT PROGRAM AND
GENE THERAPY CONSORTIUM PROGRAM
ARE TWO BOTTLES IN A WAY OF
DELIVERING DIFFERENT THERAPEUTIC
MODALITIES. RARE DISEASE -- RARE
DISEASE TRIALS. WE STAND TO
LEARN THE NEXT COUPLE OF YEARS A
LOT FROM THAT. THE FACT THAT WE
ARE DOING THOSE -- THAT WORK AT
NCATS NIH WITH OUR PARTNERS
NHGRI NINDS AND CLINICAL CENTER
AND PUBLIC PRIVATE PARTNERSHIP
OF THE BGTC PROGRAM WE HAVE AN
OPPORTUNITY TO PERHAPS REALLY
TAKE A LOOK AT HOW THOSE WORK,
WE ARE RIGHT NOW IN MORE OF THE
PRE-CLINICAL PHASE NOW AND
GETTING READY TO MOVE INTO THE
CLINIC THE NEXT COUPLE OF YEARS.
SO I THINK PROBABLY ABOUT FIVE
YEARS WE WILL BE ABLE TO WRITE
SOMETHING THAT LOOKS LIKE THIS
TO DISSEMINATE TO COMMUNITIES
THAT COULD POTENTIALLY BE
HELPFUL AS WELL NOT JUST
COMMUNITIES BUT THE BROADER
ACADEMIC MEDICAL ORGANIZATIONS
THAT ALSO PROVIDE INFRASTRUCTURE
NEEDED TO MAKE THESE MUCH MORE
ROBUST. ACROSS THE BOARD. THIS
IS A GREAT IDEA AND PJ IS TAKING
NOTE OF THAT AS WELL, WE WILL
CONSIDER THAT TOO. WE HAVE BEEN
THINKING DISSEMINATION OF
PRE-CLINICAL SPACE AND ORPHAN
DRUG DEVELOPMENT TEMPLATE, THE
RARE PEDIATRIC DESIGNATION
TEMPLATE WITH THE FDA. WE HAVE
MADE THE WORK THAT WE HAVE DONE
PUBLICLY AVAILABLE SO OTHERS CAN
LEARN FROM THAT. BUT THE
MATURITY MODEL THE SITE
PRACTICES AND READINESS ARE
STILL KEY NEEDS FOR COMMUNITIES
TO UNDERSTAND HOW THAT NEEDS TO
BE TRANSLATED TO RARE DISEASE.
SO I THINK IT IS A REALLY GOOD
POINT. THANK YOU.
>> ALL RIGHT. I THINK DO WE HAVE
DR. WEGRZYN ON THE LINE?
>> SHE IS ON THE PHONE.
>> RENEE I CAN'T SEE YOU YET BUT
SO GLAD YOU ARE HERE. IT IS MY
PLEASURE TO INTRODUCE DR. RENEE
WEGRZYN AS THE FIRST DIRECTOR OF
THE ADVANCED RESEARCH PROJECTS
AGENCY FOR HEALTH OR ARPA H AS
YOU HAVE HEARD IT CALLED. WAR
PA SHE WAS APPOINTED IN OCTOBER
11, SO ALMOST A YEAR AGO BY
PRESIDENT JOE BIDEN. SHE
PREVIOUSLY SERVED AS THE VICE
PRESIDENT OF BUSINESS
DEVELOPMENT AT GINKO BIOWORKS
AND HEAD OF INNOVATION AT
CONCENTRIC BY GEEKO SHE FOCUSED
ON APPLYING SYNTHETIC BIOLOGY TO
OUTPACE INFECTIOUS DISEASE.
INCLUDING COVID-19. THROUGH
BIOMANUFACTURING, VACCINE
INNOVATION, AND BIOSURVEILLANCE
OF PATHOGENS AT SCALE THAT, IS
WHERE THE -- A LOT OF WORK SHE
HAS DONE COMES FROM. SHE COMES
WITH EXPERIENCE WORKING FOR TWO
INSTITUTIONS THAT INSPIRED THE
CREATION OF ARPA H. AT DARPA
THEN AT IARPA AS WELL SO DARPA
IS DEFENSE AND IARPA IS
INTELLIGENCE ARPA AND PROGRAM
MANAGER AT DARPA FOR A VARIETY
OF YEARS, AND SHE HAD A
PORTFOLIO AROUND SAFE GENES
PREEMPTIVE EXPRESSION OF
PROTECTIVE ALLELES AND RESPONSE
ELEMENTS AS WELL AS VARIETY OF
WAYS WHICH TO DETECT AND FIGURE
OUT WAYS TO GENE EDIT DEVELOP
GENE EDITING TECHNOLOGIES AND
PROGRAMS. SHE RECEIVED THE
SUPERIOR PUBLIC SERVICE MEDAL
FOR WORK IN CONTRIBUTIONS AT
DARPA AND SO WE ARE REALLY
EXCITED TO HAVE HER TODAY TO
JOIN US TO TELL US A LITTLE BIT
MORE ABOUT WHAT SHE WILL BE
DOING AT ARPA H, WHAT SHE'S BEEN
DOING AND WHAT SHE WILL BE
DOING. I HOPE WE WILL ALSO TALK
A LITTLE BIT ABOUT SOME OF THE
WORK WE HAVE BEEN DONE TOGETHER.
SO IT IS REALLY DELIGHTFUL TO
SEE YOU HERE, RENEE. WELCOME TO
OUR NCATS COUNCIL. TAKE IT AWAY
>> THANK YOU SO MUCH, JONI.
REALLY APPRECIATE THE TIME TODAY
TO TALK A LITTLE BIT ABOUT ARPA
H. I WILL SAY BEEN A DELIGHT TO
WORK WITH JONI AND TEAM AND HOPE
THIS IS OUR PARTNERSHIP
CONTINUES TO GROW AND SOME OF
THE THINGS YOU HEAR TODAY I HOPE
INSPIRE YOU TO FIGURE OUT WAYS
TO WORK TOGETHER EVEN MORE. I
WILL SPEND MAYBE 15, 20 MINUTES
TALKING ABOUT THE MISSION AND
WHAT WE DO AND I THINK I HAVE AT
LEAST TEN MINUTES FOR QUESTIONS.
I LOVE THIS TO BE A DYNAMIC Q&A
SECTION. IF ANYTHING IS UNCLEAR
FEEL FREE TO INTERRUPT AND I
WILL TRY TO CLARIFY ALONG THE
WAY. SO CAN YOU SEE THE DECK?
YOU GUYS ARE TINY.
>> YES.
>> GREAT. PERFECT. SO ARPA H AS
JONI MENTIONED HAS BEEN AROUND A
LITTLE OVER A YEAR, I WAS SWORN
IN LAST OCTOB OCTOBER. LAST MAR,
2022, PRESIDENT BIDEN CALLED ON
CONGRESS TO FUND ARPA H
INITIALLY, REALLY SPEAKING WITH
THE PRESIDENT ABOUT THIS, HE HAD
A CLEAR VISION ON APPLYING THE
DARPA BUSINESS MODEL WHICH IS
MOVING VERY QUICKLY, HAVING A
LOT OF AUTONOMY AND DECISION
MAKING BUT PIN STEAD OF DOING
THAT FOR DEFENSE TO BE ABLE TO
DO THAT FOR HEALTH AND MAKE
REALLY BIG BETS IN HEALTH AND
CREATE AN AGENCY THAT CAN
SUPPORT AND DO THAT. I'M TOLD
JONI THIS BEFORE, I DON'T THINK
THERE WOULD BE AN ARPA H IF NOT
FOR NCATS FIRST WHERE YOU CAN
REALLY SHOW BY HAVING A LITTLE
BIT OF A DIFFERENT MODEL YOU CAN
REALLY ACCELERATE SOME OF
BRINGING THOSE BETTER HEALTH
OUTCOMES TO AMERICANS. SO WE
HOPE TO REALLY CAPITALIZE ON THE
WORK THAT NCATS HAS DONE BUT
ACCELERATE FURTHER WITH BROADER
MMANDATE AS WELL. SO OUR MISSIO,
ONE FIRST THING WALKING INTO
ARPA H WAS TO CREATE A MISSION
STATEMENT. TO SPEND A SECOND,
ACT RATE ACCELERATE BETTER
HEALTH OUTCOMES FOR EVERYBODY. A
LOT OF GOOD WORK HAPPENING AT
UNIVERSITY AND PRIVATE SECTOR,
IF WE WAITED 20, 25 YEARS THE
WORK WE INTEND TO FUND WOULD
BECOME A REALITY BUT WE ARE VERY
IMPATIENT AND WE DON'T WANT TO
WAIT. SO THROUGH OUR INVESTMENTS
ARPA H WANTS TO ACCELERATE
BRINGING A LOT OF THOSE THINGS
TO THE MARKET AND INTO THE REAL
WORLD TO IMPACT HEALTH OUTCOMES.
SO WE DON'T MEASURE OUR PROGRESS
BY NUMBER OF COMPANIES WE LAUNCH
OR NUMBER OF PAPERS WE PUBLISH,
BUT ACTUAL MEASURABLE CHANGES
AND QUALITY OF LIFE OF AMERICANS
AND ALL AMERICANS, IT SHOULD BE
FOR EVERYONE. SO ACCESSIBILITY
COSTS EQUITY ARE REALLY BIG
FOCUS OF THE WORK THAT WE DO. SO
HOW CAN WE GO AFTER THESE BIG
BETS THE PRESIDENT HAS CALLED US
TO DO? PART OF THAT IS THROUGH
OUR AUTHORITIES AND HOW WE WERE
SET UP. SO THE FIRST THREE
MONTHS AT MY JOB AT ARPA H WERE
SPENT TIME ON THE HILL WITH
MEMBERS OF CONGRESS TO TALK
ABOUT THE THINGS THAT WE CAN BE
UNCOMPROMISING ON. OUR UNIQUE
STRUCTURE IS VERIFY AN INSTITUTE
AT NIH BUT WE ARE WITHIN NIH SO
I CAN LEVERAGE A LOT OF
EXPERTISE INSTITUTES LIKE NCATS.
BUT WE ARE OUR OWN AGENCY, OUR
OWN BUDGET AND I DON'T REPORT TO
THE ARPA H -- I'M SORRY TO THE
NIH DIRECTOR. I REPORT TO
SECRETARY BECERRA. SO THAT ALLOW
US TO NOT ONLY HAVE AUTONOMY
HAVING A DIFFERENT BUDGET BUT
ALSO A DIFFERENT REPORTING
STRUCTURE AND DIFFERENT AGENDA.
WE DON'T HAVE ANY INTERNAL
RESEARCH LABS SO JUST A FUNDING
AGENCY. AND WE GENERALLY WILL
FUND CONTRACTS, NOT GRANTS, SO
COOPERATIVE AGREEMENTS, BECAUSE
WE WANTS TO A SEAT AT THE TABLE,
WANT TO BE PART OF THE DECISION
MAKING PROCESS AND AS THE MARKET
SHIFTS AND NEEDS CHANGE WE WANT
OUR CONTRACTS AND TOOLS TO BE
ABLE TO CHANGE AS WELL. OUR
INITIAL APPROPRIATION WE HAD AN
INITIAL TWO AND A HALF BILLION
DOLLARS THAT WE LAUNCHED. WE
HOPE TO GROW THAT BUDGET OVER
TIME. THESE AUTHORITIES SHAPE
FEATURES OF WHAT IS ARPA H. I
HAVEN'T MENTIONED PROGRAM
MANAGERS YET. THAT IS A BIG PART
OF THE AGENCY SO THROUGH OUR
AUTHORITIES WE CAN HIRE PROGRAM
MANAGERS FOR TERM LIMITS THREE
YEARS TO START, UP TO ADDITIONAL
THREE YEARS, AND THESE ARE FOLKS
THAT WE HIRE NOT BECAUSE OF
THEIR BRILLIANT MINDS, THEY ARE
CLEAR DOERS IN THEIR FIELDS BUT
BRING ARPA H A PROBLEM THEY WANT
TO SOLVE. SO I'M WHEN SELECTING
A CANDIDATE IT IS NOT JUST
BECAUSE THEY ARE AMAZING PERSON
BUT ALSO HAVE A PROBLEM, THAT I
THINK ARPA H INVESTMENT GIVE
ASIP METRICAL ADVANTAGE TO
HEALTHCARE IF WE ABLE TO MOVE
THE NEEDLE ON THAT GIVEN
PROBLEM. T I MENTION ACCELERATED
CONTRACTING AND EVEN THE
FLEXIBILITY IN HIRING WHEN YOU
FIND PROGRAM MANAGERS YOU WANT
TO BRING THEM HON IMMEDIATELY SO
WE HAVE ADMINISTRATIVELY
DETERMINED POSITIONS AS WELL AS
DIRECT HIRING AUTHORITIES SO WE
DON'T USE USA JOBS. WE CAN GO
DIRECTLY TO A PERSON HIRE THEM
BUT ALSO RELATIVELY COMPETITIVE
SALARY THAT IS OUT -- OUTSIDE OF
THE GS SCALE GOVERNMENT. SO
THESE PROGRAM MANAGER IT IS
PROGRAMS THEY LAUNCH AND AGENCY
AS A WHOLE, WE SERVE THE HEALTH
ECOSYSTEM FROM OUR CUSTOMERS IN
PUBLIC AND HEALTHCARE PROVIDERS
TO PERFORMERS AND ACADEMIA AND
INDUSTRY, BUT ALSO STAKEHOLDERS
LIKE NIH BUT ALSO FDA AND CMS
AND OTHERS WE KNOW IN ADDITION
TO AMAZING INNOVATION TECHNOLOGY
TO BRIDGE THAT GAP TO THOSE
OUTCOMES. WE NEED TO MAKE SURE
IT IS REGULATED AN PAID FOR AS
WE MOVE FORWARD. SO I TALKED
ABOUT THESE ORGANIZATIONAL
ATTRIBUTES LIKE THE PROGRAM
MANAGERS FOCUSING ON THESE
PROGRAMS AND RADICAL CHANGES
USHERED IN, FOR THIS GROUP IN
PARTICULAR IT IS IMPORTANT TO
MENTION AUTONOMY WE HAVE IN OUR
DECISION MAKE SO THROUGH
AUTHORITIES WE CAN GIVE PROGRAM
MANAGER IT IS SOLD DISCRETION TO
SELECT AND FUNDS PROJECTS SO
THIS IS DIFFERENT THAN USING A
STUDY SECTION OR A COMMITTEE. TO
MAKE THOSE DECISIONS WE WANT TO
TAKE ON A LOT OVARIES INCOME THE
WORK THAT WE DO, TECHNICAL RISK
NOT EXECUTION. AND WE DO THAT BY
HAVING A SINGLE THREAD OF
DECISION MAKER P P WHICH IS
UNIQUE FOR OUR MODEL. TAKEN
TOGETHER WHEN PROGRAM MANAGERS
COME ON BOARD THEY BRING A
PROGRAM IDEA WITH THEM THEY
LAUNCH RIGHT AWAY AND ONCE A
YEAR, I CALL ON THEM TO LAUNCH
ANOTHER PROGRAM. IF YOU ARE HERE
THREE YEARS YOU LAUNCH THREE
PROGRAMS. THOSE CAN BE DIFFERENT
FROM ONE ANOTHER BY PORTFOLIO AT
DARPA AS JAMIE MENTIONED WAS
DIVERSE. SO WE EXPECT THOSE
PROGRAM MANAGERS TO BRING IN
THOSE NEW PROGRAMS AND YOU GO
FLY WHEEL OF NEW STAFF, NEW
PROGRAMS WE INVEST IN. SO THE
PORTFOLIO WHAT IT FUNDS IS
PROGRAM MANAGERS IS DYNAMIC AND
CHANGES FREQUENTLY. SO THE
PORTFOLIO WE FUND THIS YEAR I
WILL TALK ABOUT THE INVESTMENTS
YOU MADE IN CANCER MOON SHOT, WE
ANNOUNCED A BIOFABRICATED HEART
PROJECT. SO THESE ARE DIVERSE,
DOLLARS ARE NOT ALIGNED TO A
SINGLE DISEASE THAT ALLOWS US TO
FUND AREAS WE THINK HAVE
GREATEST IMPACT. SO THESE OUR
FIRST COHORT OF PROGRAM
MANAGERS, WE HAVE 15 BETWEEN
THOSE IN HOUSE AND COMMITTED TO
BRING ON BOARD BOARD. I LOVE TOK
GROUPS LIKE THIS THINK OF ONE OR
TWO PEOPLE IN YOUR NETWORK, A
GREAT PROGRAM MANAGER THAT HAVE
THESE UNCOMMON PEOPLE WITH
COMMON TRAITS, LIKE HAVING THE
SERIOUS DRIVE YOU THINK LIKE A
CEO BUT THEY ALSO HAVE TECHNICAL
HONESTY SO A CEO HAS TO KNOW
WHAT THE GOAL IS, AT THE END, IF
THINGS ARE NOT WORKING OUT TO
PIVOT RESOURCE SO THAT IS
DIFFERENT ABOUT ARPA H AS WELL,
IF A PROJECT IS NOT WORKING WE
WILL PIVOT RESOURCES, WE MIGHT
NOT AWARD A PHASE 2 OF A PROJECT
IF ANOTHER LOOK MORRIS
PROMISING. THE FOLKS WE HIRE CAN
BE DIFFERENT STAGE OF THEIR
CAREER, WHETHER EARLY STAGE, THE
WORLD IS YOUR OYSTER TO STATUS
QUO CHALLENGES THAT ARE MID
CAREER BUT HAVE A BLOCKER TO SAY
MY FIELD WOULD BE TOTALLY
DIFFERENT IF WE HAD X. I SAY OUR
FIRST PROGRAM MANAGER OF US FELL
INTO THIS CATEGORY HE IS A
SURGEON AND DID THE MATH AND
SAID I CAN DO A THOUSANDS, 2,000
KNEE TRANSPLANTS OVER THE COURSE
OF CAREER OR COME TO ARPA H AND
REVERSE OSTEOARTHRITIS, IS
THAT'S WHAT I WANT TO DO AND THE
SCALE I WANT TO WORK ON. SO WE
HAVE A CHALLENGE WITH DIVERSITY,
THE PROGRAM MANAGERS WE ARE
HIRING STEM DOES GENERALLY SO
THINK ABOUT PLACE YOU THINK
ABOUT DIVERSE NETWORK WE ALSO
WANT TO SERVE COMMUNITIES THAT
REFLECT ALL OF AMERICA. SO WE
EDNA TO CLOSE THAT GAP BETTER.
WHEN WE FRAME OUR QUESTIONS OF
HOW WHICH WORK ON A PROBLEM AT
ARPA H WE TACKINESSRY OPERATION
FROM DARPA. WE TAKE INSPIRATION
FROM DARP. THEY CAN THESE
QUESTIONS AND FUND ANY PROJECT
YOU ARE WORKING ON FROM EXPERT
TO LAY PERSON. WHAT PROBLEM ARE
YOU TRYING TO SOLVE? HOW IS IT
DONE TODAY? NEW IN YOUR
APPROACH, THIS IS WHERE THAT
DEEP TECHNICAL INSIGHT, ONCE THE
PAPER THE PATENT THE DATA THAT
CONVINCES YOU GAME CHANGING AT
THIS TIME. WHO IMPACT, WHAT ARE
RISKS, HOW LONG IT WILL TAKE AND
COST. BECAUSE OF THESE BIG BETS
YOU MIGHT NOT SEE WHAT THEY
DELIVER UNTIL 10, 15 YEARS DOWN
THE ROAD. WHAT ARE THE MIDTERM
EXAMS, YOU KNOW YOU ARE ON THE
RIGHT TRACK A YEAR FROM NOW, TWO
YEARS FROM NOW. THEN WE ADDED
TWO ADDITIONAL QUESTIONS, ONE
THAT REALLY WANT TO ENSURE
EQUITABLE ACCESS FOR ALL PEOPLE,
ADDRESSING COST ACCESSIBILITY
USER EXPERIENCE, WE HAVE TALKED
WITH RARE DISEASE GROUPS AND WE
ARE ASKING, WE DON'T WANT TO
CREATE THE NEXT MILLION DOLLARS
SINGLE DOSE THERAPY, IF YOU WANT
A SOLUTION TO ARPA H TO MOVE
FORWARD HOW ARE YOU ADDRESSING
SUPPLY CHANGE SO WE CAN THOUSAND
X, 10,000 X REDUCE THAT COST
GOING FORWARDS. THE LAST IS
ABOUT BUILDING TRUST WITH
COMMUNITIES. WE DESIGNED WITH
CUSTOMER EXPERIENCE IN MIND,
CREATE TOOLS PEOPLE WANT TO
ENTHUSIASTICALLY ADOPT, WE DON'T
WANT TO PRESCRIBE SOLUTIONS, WE
WANT TO WORK WITH COMMUNITIES
AND MAKE SURE THERE'S NO
MISPERCEPTION ENVISION JOB
CREATING FUTURES FOR HEALTH. IS
IT FUTURE YOU WANT TO LIVE IN
AND HOW DO YOU COMMUNICATE WITH
THAT FUTURE IS, SO THAT I IT IS
NOT SCARY AND WE CAN BRING
PEOPLE ALONG THE RIDE. THE NEXT
ARE CARTOONS HOW WE WORK ON THIS
MODEL SO BRINGING THE PROGRAM
MANAGER AND IDEA TOGETHER, I
GIVE THEM A BANK ACCOUNT TO HIT
THE GROUND RUNNING AND LAUNCH A
PROGRAM, THEY SOLICIT IDEAS FROM
THE COMMUNITY SO THEY FUND TEAMS
OF PERFORMER THAT HAVE DIFFERENT
WAY TO APPROACH A SOLUTION. TO
THAT PROBLEM SO PORTFOLIO OF
RISK AS WELL IN PERFORMERS THAT
ARE SELECTED FORGIVEN PROGRAM.
SO CAN BE HIGH RISK, SOME MIGHT
MITIGATE IT A LITTLE BIT. THEN
THE PROGRAM MANAGER THIS IS WHY
WE HAVE CONTRACTS COOPERATIVE
AGREEMENTS AND OTHER TRANSAC
TRANSACTIONS BECAUSE EVEN
SOMETIMESES ON A DAY-TO-DAY
BASIS INTERACT WITH PERFORMERS
AND GUIDE THE RESEARCH AND SERVE
AS PARTNER. SO OVER THE COURSE
OF THE PROJECT, THEY ARE NOT
ONLY PROVIDING SUPPORT THEY WANT
THEM TO BE SUCCESSFUL AND REACH
THAT END DELIVERABLE BETTER
HEALTH OUTCOMES. IF A PERFORMER
FOR WHATEVER REASON, MAYBE THERE
IS LAW OF PHYSICS THEY COME UP
AGAINST AND THEIR FUNDING IS NOT
EXTENDED, THE PROGRAM MANAGER
WILL MAKE THOSE HARD DECISIONS
OVER THE COURSE OF A PROJECT
BECAUSE THEIR GOAL IS TO GET AT
LEAST ONE GROUP ACROSS THE
FINISH LINE. WE HAVE A REAL
COMMITMENT TO TRANSITION, WHAT I
MEAN BY TRANSITION IS LIKE
TRANSITION OUT OF THE AGENCY.
SOME OF THIS WILL BE
TRANSLATIONAL, NOT ALL OF IT IS.
BUT ARPA H FUNDING IS A
TRANSACTION. WE ARE HERE TO
DEMONSTRATE AND SHOW THAT
SOMETHING IS POSSIBLE, REMOVE A
LOT OF UNCERTAINTY AROUND A
CONCEPT. IF SUCCESSFUL MAYBE
NCATS PICKS IT UP OR MAYBE
PRIVATE SECTOR PICKS UP. MAKE IT
GOES OVER TO A CLINICAL TRIAL,
ALL THOSE ARE VALID
POSSIBILITIES. BUT WE WILL NOT
BE A FOREVER FUNDER OF A
PROJECT. WE ARE HERE TO BUY DOWN
THAT RISK. THIS IS ONE OF THE
WAYS PEOPLE ARE LIKE, YOU ARE
INSPIRED BY DARPA BUT DIFFERENT
SO WHAT ARE SOME OF THE WAYS YOU
ARE DIFFERENT? THIS IS ONE OF
THE MAJOR WAYINGS THAT WE ARE
DIFFERENT. IS -- WAYS WE ARE
DIFFERENT IS HOW TO TRANSITION
AND ACCELERATE. SO IF YOU ARE AT
DARPA, PROJECT SUCCESSFUL,
DEPARTMENT OF DEFENSE WILL PONY
UP LOTS OF MONEY TO BE THE
DOWNSTREAM ADVANCE DEVELOPER TO
MAKE THESE A REALITY. IF I
DEVELOP A SATELLITE AT DARPA DOD
WILL MAKE THAT AND PRODUCE IT
LONG TERM. WE DON'T HAVE THAT IN
THE HEALTH ECOSYSTEM. WE HAVE
HHS THAT EXISTS BUT THEY ARE NOT
A MANUFACTURER, THEY ARE ALSO
NOT A CENTRAL BODY THAT FUNDS
HEALTHCARE IN AMERICA. WE HAVE A
FEDERATED SYSTEM THAT IS ACROSS
ALL 50 STATES, LOOKS DIFFERENT
DEPENDING THE STATE YOU ARE IN.
THIS OFTEN IN THIS IS TRYING TO
CREATE THE -- OFFICE SRI TRYING
TO CREATE THE ECOSYSTEM. WE WORK
WITH PROGRAM MANAGERS BEFORE
THEY LAUNCH PROGRAMS TO MARKET
ASSESSMENT WITH VENTURE
CAPITALISTS THAT ARE PART OF THE
PARTNERSHIP TEAM TO SAY OKAY
THERE IS A MARKET IN THIS PLACE
OR THERE IS NOT AND MAKE SURE WE
ARE BUYING DOWN THAT DOWNSTREAM
INVESTMENT RISK TOO SO WE WANT
TO DERISK THINGS NOT ONLY THE
TECHNOLOGY BUT WHAT DO INVESTORS
NEED TO SEE SO THEY WILL PICK
THIS UP. THIS IS ALL ALONG THE
COURSE OF PROGRAM, WE ARE
WORKING CLOSELY WITH TRANSITION
OFFICE BECAUSE WE DON'T WANT IT
TO BE A LAST DAY CONVERSATION.
WE NEED TO BE THINKING ABOUT IP,
ET CETERA. MAYBE EVEN SPINNING
OUT A COMPANY, WE WILL SUPPORT
THAT PROCESS AS WELL. OUR
PROGRAM MANAGERS DO COME TO THE
TABLE WITH AN IDEA OF A PROGRAM
THEY WANT TO FUND AND MOVE
FORWARD WITH. I HAVE IDENTIFIED
FOUR AREAS IN HEALTH. FOCUS
AREAS WE ARE TRYING TO CREATE
BUCKETS OF IDEA OR UMBRELLAS WE
OPERATE. THESE ARE BECOMING
MISSION OFFICE IT IS PROGRAM
MANAGER TEAMS DEVELOP UNDER.
HEALTH SCIENCE NEURO, EXPANDING
WHAT IS TECHNICALLY POSSIBLE,
ONE RECENT ANNOUNCEMENTS THE
CARROT PROJECT, REALLY DIRECT
MODULATION OF THE IMMUNE SYSTEM,
YOU SAYING DEAD CAST MODULATORS
THAT UP AND DOWN REGULATE GENES
INVOLVED IN IMMUNE RESPONSE. WE
HAVE THREE APPLICATIONS FOR
CANCER, LONG COVID AND FOR
LUPULUPUS. SO THOSE ARE THREE
DIFFERENT DISEASES RAISING A
PLATFORM TECHNOLOGY TO
DEMONSTRATE THAT. SO THIS IS THE
OFFICE IS THE ONE THAT WILL
BRING THOSE PLATFORMS FORWARD.
SCALABLE SOLUTIONS. THIS IS NOT
JUST MANUFACTURING AT SCALE, BUT
MEETING THE SCALE OF MEETING
AMERICANS WHERE THEY ARE AT IN
THEIR HOMES THAT LAST MILE OF
GETTING SOMETHING OUT OF THE LAB
INTO THE REAL WORLD BUT THEN
INTO A RURAL SETTING THAT IS
HUNDREDS OF MILES FROM
CENTRALIZED LOCATION. THESE ARE
TYPES OF INNOVATIONS WE LIKE TO
ADDRESS WITH SCALABLE SOLUTIONS
PROACTIVE HEALTH IS KEEPING
PEOPLE FROM BECOMING PATIENTS IN
THE FIRST PLACE. DIAGNOSTIC,
DETECTION, BEHAVE I DON'T RECALL
MENTAL HEALTH. ALL FALL UNDER
THIS OFFICE. THEN LAST BUT NOT
LEAST RESILIENT SYSTEMS IS WE
JUST SHOWED LAST THREE YEARS WE
DON'T HAVE A RESILIENT
HEALTHCARE SYSTEM, WHAT ARE THE
INVESTMENTS WE ARE MAKING NO NON
DIGITAL SOLUTIONS INNOVATION
THAT WE CAN BRING FORWARD. WHAT
IS GREAT ABOUT BEING NEW
GOVERNMENT AGENCY IS WE DON'T
HAVE TO TAKE ON ANY LEGACY DATA
SYSTEMS. WE CAN CREATE OUR OWN
AND MAKE IT RELEVANT TO MOVE AT
SPEED OF PRIVATE SECTOR, ALSO
SOMETHING WE ARE CRYING TO
CREATE. OUR FIRST PROGRAM WE
LAUNCHED IS CALLED NITRO. FROM
ROSS, THE SURGEON THAT I
MENTIONED. AND SO THIS IS JUST
TO GIVE YOU AN IDEA HOW YOU CAN
GET REALLY ULTRA FOCUSED ON
SOLVING A PROBLEM. SOL HE IS
ASKING QUESTIONS, WHAT IF OUR
JOINTS COULD HEAL THEMSELVES?
THIS IS A DEEPLY TECHNICAL FIVE
YEAR PROGRAM FOCUSED ON NO,
MA'AM MALL BASE BONE AND
CARTILAGE REGENERATION BUT
REPLACEMENT OF JOINTS FROM HUMAN
CELLS COMPLETELY GROWN IN
LABORATORY. OVER THE COURSE OF
THE PROGRAM THIS POO FOCUSES ON
NOT JUST DEMONSTRATING
MANUFACTURING OF THOSE CELLS BUT
PRE-CLINICAL WORK AND CLINICAL
WORK THAT WILL BE REQUIRED HERE
IMPORTANTLY WE HAVE A STRONG
LEVER AS A FUNDER WHERE THAT WE
CAN REQUIRE THIS CLINICAL TRIAL
ASSOCIATED WITH THIS PROGRAM
REFLECTS THE DEMOGRAPHICS OF
DISEASE OF THOSE THAT ARE
AFFLICTED. SO THIS IS A HARD
METRIC TO MEET BUT I WILL TALK
ABOUT ARPA H, THE NEW NETWORK WE
TRYING TO CREATE TO ACCELERATE
CLINICAL TRIALS AND GET THAT
ENROLLMENT UP AND RUNNING FASTER
THAN WE NORMALLY WOULD. IF YOU
HAVE BEEN TRACKING
ANNOUNCEMENTS, WE HAVE HAD NOW
QUITE A FAINEANCESMENTS IN THE
MOON SHOT REALM -- QUITE A FEW
ANNOUNCEMENTS IN THE CANCER
REALM WE ARE NOT MANDATED TO
WORK ON IT BUT FELT COMPELD BY
SOME OF THE APPLICATIONS THAT
HAVE COME IN AROUND MOON SHOT WE
THINK WE CAN START TO DELIVER ON
WHAT THE PRESIDENT HAS
CHALLENGED OUR NATION TO DO. TO
ADVANCE SO CANCER MOON SHOT
GOALS. PROGRAMS LIKE PRECISION
SURGICAL INTERVENTION TO ALLOW A
DOCK IN THE OPERATING ROOM TO BE
ABLE TO LOOK INTO THE SURGICAL
CAVITY AND SEE THE MARGINS OF
TUMOR NO LONGER PATHOLOGIST CUT
TUMORS RUN DOWN THE HALL AND
SAY, THESE ARE ONE AND DONE
SURGERIES, IN THIS PROGRAM. AND
BIOMEDICAL DATA FABRIC. THIS IS
TRYING TO BREAK DOWN SOME OF THE
SILOS OF THOUSANDS OF RESEARCH
CLINICAL INSTITUTES AROUND THE
COUNTRY WHERE DATA IS HOUSED
TODAY IN DIFFICULT TO ACCESS. SO
HOW DO WE CREATE A ROSETTA STONE
OF THE NETWORK THAT ALLOW US US
TO POOL AND LEARN ACROSS THOSE
DATA STREAMS TO ADDRESS DISEASE
LIKE CANCER BUT TO BE PLATFORM
FOR OTHER DISEASES. I MENTION
THE AMINO GENE PROGRAMMING AT
CURE IT. AND I WILL LET YOU READ
THE OTHERS BUT THIS IS AN
EXAMPLE OF VERY, VERY DIVERSE
PROJECTS TO ADDRESS SOME OF
THESE TOUGH CHALLENGES THAT ARPA
H WILL MAKE AN INVESTMENT IN,
DERISK THESE AREAS AND THEN
TRANSITION THOSE OUT OF THE
AGENCY. (INAUDIBLE) ANNOUNCED
ARPA H AS WELL THIS IS A NOD TO
DARPA HISTORY. ARPA NET STARTED
AT DARPA AS A FOUR NODE NETWORK
TO PRECURSOR TO WHAT BECAME
KNOWN AS THE INTERNET. SO
LOOKING AT THAT INSPIRATION WE
ARE STARTING OUR OWN ARPA NE NE.
WE WANT TO E CREATE
INTERCONNECTED HEALTHCARE
INNOVATION NETWORK FOR THE
UNITED STATES TO DO THINGS LIKE
BRING PEOPLE CLOSE TOGETHER AND
THESE ARE STATUTORY REQUIREMENT
THAT CONGRESS GAVE US TO BE IN 3
GEOGRAPHIC SITES AROUND THE
COUNTRY. SO THIS IS OUR ATTEMPT
AT RECOGNIZING THAT THERE AREN'T
THREE GEOGRAPHIES IN THE COUNTRY
THAT REPRESENT ALL AMERICAN
POPULATIONS SO WE WANT TO USE
THIS AS HUB AND SPOKE NETWORK TO
RECRUIT ALL 50 STATES TO BE PART
OF THIS NETWORK. THIS IS
INCREDIBLE CONVENING POWER OF
HAVING A NETWORK TO BRING
INVESTORS INDUSTRY ACADEMIC
GROUPS, COMMUNITIES TOGETHER IN
ORDER TO SOLVE HARD PROBLEMS. TO
DO SO IN INCLUSIVE WAY. ARPA NET
H THIS NETWORK CREATING ANYONE
CAN PARTICIPATE, NO PAY TO PLAY.
TO GET FUNDED THROUGH THIS
NETWORK FIRST PART OF THE
COMMUNITY IS THE LAYER BUT TO BE
FUNDED ALL THIS WORK IS DONE
THROUGH OTHER TRANSACTIONS WITH
THE SEVEN DAY TARGET. SO IMAGINE
YOU A COMMUNITY HOSPITAL THAT
DOESN'T KNOW HOW TO WORK WITH
THE FEDERAL GOVERNMENT. WE WANT
TICK MA IT SUPER SIMPLE. WE WANT
TO WORK WITH YOU AND WE DON'T
WANT US BEING THE GOVERNMENT TO
HINDER THAT. SO THIS IS A
RESOURCE FOR ALL DARPA ARPA H
PROGRAMS BUT ALSO FOR NCATS AND
NIH AND OTHER HHS BROTHER AND
SISTER AGENCIES. IMPORTANTLY,
THESE CONNECTIONS WILL ALLOW US
TO GET CLOSER TO THOSE CUSTOMERS
AND TRANSITION THINGS OUT OF
ARPA H AND THEY THEMSELVES
MENTIONED ROSS HAS DIVERSE
CLINICAL TRIAL HE IS TRYING TO E
ENROLL TO HELP US DO THAT. SO
BIG ANNOUNCEMENTS THIS WEEK OUR
STAKEHOLDER OPERATIONS HUB IN
THE NATIONAL CAPITAL REGION WE
NEED TO STAY CLOSE TO CMS FDA TO
TRANSITION CAPABILITIES. BUT
DALLAS AND GREATER TEXAS IS
GOING TO SERVE AS CUSTOMER
EXPERIENCE HUB. THERE IS
PROBABLY 25 DIFFERENT TOWNS AND
CITIES IN TEXAS REPRESENTED
HERE, POPULATIONS ALL ACROSS
URBAN AND RURAL SETTINGS BUT
IMPORTANTLY RECRUITING IN 50
STATES AS WELL. OUR LAST HUB
INVESTOR CATALYST HUB TO HELP
GET TO INVESTORS WHO WILL BRING
CAPITAL DOWNSTREAM TO ADVANCE
ARPA H PROGRAMS WILL BE BASED
OUT OF GREATER BOSTON AREA. THEN
LAST BUT NOT LEAST FOR THIS
GROUP, THINKING ABOUT WAYS TO
WORK WITH THIS, I WILL MENTION
BECOMING PROGRAM MANAGER, YOU
CAN TWO TO OUR WEB FORM NOT USA
JOBS IS A NICE GOOGLE FORM ON
THE WEBSITE TO APPLY AND OPEN
BROAD AGENCY ANNOUNCEMENTS SO
WHILE MOST EFFORTS ARE PROGRAMS
LAUNCHED BY PROGRAM MANAGERS WE
HAVE A WAY, IF YOU HAVE A GREAT
IDEA THAT MAYBE WE HAVEN'T
THOUGHT OF WE WANT TO HEAR FROM
YOU. WE MADE IT A THREE PAGER
THAT WHICH WANT TO HEAR THE
IDEAS BUT -- WE WANT TO HEAR
THOSE IDEAS BUT NOT SPEND A LOT
OF TIME TO GET FEEDBACK AND SEND
THOSE IDEAS TO THE OPEN BAA. I
LIKE TO END ON THIS I FEEL LIKE
EVERY WEEK AT ARPA H WE
ACCOMPLISH SO MUCH MORE THAN THE
WEEK BEFORE, WE HAD BIG
ANNOUNCEMENTS GROWING OUR
NETWORK. THE FUTURE WE WANT TO
CREATE IS HARD TO IMAGINE NOW IN
HEALTH AND ONE OF THE GROUP THAT
IS REALLY RESPONSIBLE FOR
LEADING THE CHARGE THERE. WITH
THAT I WILL STOP THE SCREEN
SHARE. HAPPY TO TAKE ANY
QUESTIONS AND AGAIN, LOTS OF
GRATITUDE TO JONI AND TEAM FOR
INVITING ME HERE TODAY.
>> RENEE, THANK YOU SO MUCH,
WONDERFUL OVERVIEW, I WOULD LIKE
TO OPEN UP TO ANY QUESTIONS FROM
OUR COUNCIL MEMBERS.
>> CONGRATULATIONS AND EXCITED
TO SEE THE SPEED WHICH YOU
LAUNCHED OFF NOT EXCITED TO SEE
THE FIRST PROGRAM MANAGER COME
ON. AS YOU MENTIONED IN THE LAST
SENTENCE SIX OUT OF EIGHT ARE
WHITE MEN AND SPEAKING AS WHITE
MAN MYSELF I BENEFIT A LOT FROM
DIVERSITY AND LEADERSHIP WHO
THINKS DIFFERENT INCOMES FROM A
DIFFERENT HISTORY AND DISRUPTIVE
INSIGHT WHOSE HAVE LEVELS OF
DIVERSITY HERE IN THE US.
THAT'S ONE OF THE REASONS I CAME
AND VERY HAPPY TO CONTRIBUTE TO
THE PROGRESS IN THIS GREAT
NATION OF OURS. SECOND COMMENT,
DARPA IS VERY SUCCESSFUL BECAUSE
AS YOU MENTIONED THIS IS A
FANTASTIC KITCHEN OF GREAT IDEAS
AND DOD TAKES IT AND PUSH IT
THROUGH WITH FORCE. HERE IN THE
HEALTHCARE SECTOR WE DON'T HAVE
THAT. AND VENTURE CAPITAL IS THE
SOURCE OF FINANCE TO BRING IN.
BUT VENTURE CAPITAL FINANCES
HAVE SERVED THOSE RESOURCE WELL
IN OUR COUNTRY. AND NOT ALWAYS
NOT WELL RESOURCED AND
UNDERSERVED POPULATIONS IS A LOT
OF WHERE DISEASE BURDEN IS. SO A
MAJOR CONCERN IF ARPA H RELIES
ON FINANCING TO MOVE FORWARD,
THAT MIGHT INADVERTENTLY RESULT
IN YOUR INVESTMENTS AGAIN GOING
TO THOSE WHO ARE IN NEED. I
DON'T HAVE A SOLUTION TO THAT
BUT THAT MIGHT IN ITSELF BE A
PROBLEM FOR -- TO PUT IN FRONT
OF.
>> YOU HIGHLIGHT A REALLY
IMPORTANT PROBLEM WHICH IS GOING
BACK TO THE HOW AM I RIGHT FROM
THE GET GO SAY DRESSING THAT
COST AND ACCESSIBILITY. EVEN IN
THE DESIGN OF OUR PROGRAMS, IF
ROSS MAKING SURE HIS CLINICAL
TRIAL REPRESENTS RAPID DISEASE
IS ACTUALLY IMPACTED BY
OSTEOARTHRITIS, THE SIDE
PROGRAM, PRECISION SURGICAL
INTERVENTIONS, THE PROGRAM
MANAGER IS REQUIRING THAT
SURGICAL DEMONSTRATION TO NOT
ONLY BE CENTER OF EXCELLENCE,
SURE WE WILL GET THOSE, BUT IN A
RURAL SETTING WHERE YOU DON'T
HAVE 5G, HOW DO YOU START TO
ACTUALLY APPLY THAT THERE. L SO
IT IS VERY INTENTIONAL AND A
REQUIREMENT OF OUR PROGRAMS.
FOR VCs WITH OUR INVESTOR
CATALYST HUB IT IS NOT JUST YOUR
TYPICAL, NOT GOING TO NAME
ANYBODY BUT YOU CAN PROBABLY TOP
TEN THINK OF YOUR VC AND HEALTH
NAMES. SURE THOSE FOLKS COME
AROUND THEY HAVE NO TROUBLE
FINDING INVESTMENTS. WHAT WE ARE
LOOKING FOR ARE INVESTORS THAT
HAVE RETURN INVESTMENT NOT ONLY
IN FINANCING BUT IN THE
COMMUNITIES THEY SERVE. SO OUR
PRIME FOR THAT EFFORT IS VENTURE
WEALTH WHO HAS REALLY GREAT
TRACK RECORD OF DELIVERING
SOLUTIONS TO UNDERSERVED
COMMUNITIES AND BEYOND. POINT
TAKEN, WE ARE NOT -- WE
DEFINITELY ARE NOT CREATING A
PIPELINE TO GO TO VC FUNDING
THAT IS NOT THE POINT BUT WE
KNOW THEY HAVE TO HAVE A SEAT AT
THE TABLE AND WE ARE TRYING TO
GUIDE THEM DOWN A PATH THAT
LOOKS AT THAT EQUITY AND ACCESS.
THE OTHER THING I WILL MENTION,
WE HAVE ONE UNIQUE AUTHORITY I
THINK NCATS MAY HAVE THIS AS
WELL BUT DON'T QUOTE ME, I
BELIEVE THAT'S JONI. WE CAN --
WE HAVE TO SORT OF REIMBURSE FDA
DIRECTLY. AND WE HAVE BEEN
WORKING WITH THE COMMISSIONERS
OFFICE, WE PUT AN RFI OUT TO THE
PUBLIC AND TO GET IDEAS AS WELL.
HOW CAN WE USE THIS AUTHORITY?
LET'S BE CREATIVE ABOUT IT. AND
SOME OF THE THINGS THAT HAVE
COME UP ARE FOR EXAMPLE, IF WE
HELP DEVELOP A DRUG MAYBE
IMMUNOMODULATOR, COULD WE, IF WE
ENSURE A COST AT A CERTAIN LEVEL
OR ACCESS TO COMMUNITIES OF
NEED, WE COULD GIVE THE PERSON
THAT IS APPLYING FOR THE
REGULATORY APPROVAL PRIORITY
ACCESS TO THE MARKETPLACE OR
FIRST ACROSS THE FINISH LINE FOR
THAT REGULATION BUT TO MEET
THOSE GOVERNMENT NEEDS OR HAVE
PRECOMPETITIVE DATA SHARING SO
TRYING TO USE TOOLS BESIDES
MONEY TO INCENTIVIZE GOOD
BEHAVIOR. IF THIS GROUP
ESPECIALLY RARE DISEASE SIDE
UNDERSERVED COMMUNITIES IF
THERE'S OTHER THINGS YOU CAN
THINK OF THAT WE CAN TRY LIKE WE
WANT TO BE A PLACE TO EXPERIMENT
IN THAT WAY AS WELL. SORRY,
LONG WIND BUD REALLY PASSIONATE
ABOUT IT AND CARE A LOT ABOUT
IT. BUT TO YOUR POINT, NOT AN E
SE QUESTION TO WANT TO BRING THE
MOST CREATIVE IDEAS THERE.
>> PAULA, DID YOU HAVE A
QUESTION? PAULA AND ANNIE THEN
MAYBE WE'LL STOP AFTER AND IN A.
>> VERY EXCITI EXCITING. YOU TAT
HAVING A RURAL HOSPITAL. HOW DO
YOU ENVISION TAKING THE
TREATMENTS TO THOSE RURAL
COMMUNITIES THAT MAKE UP TO A
FIFTH OF THE POPULATION BUT VERY
UNDERSERVED?
>> YEAH. THIS IS LIKE WE
PRESCRIBED THAT IT HAS TO BE IN
A RURAL SETTING AND WE ARE
REQUIRING PERFORMERS TO BRING US
THEIR SOLUTION. O SO THIS IS A
PLACE WE WILL SET A BAR THEN
EXPECT THE COMMUNITY TO COME TO
THAT. THAT IS AN ACTIVE PROPOSAL
PROCESS RIGHT NOW WE HAVEN'T
SELECTED THE TEAMS YET BUT WHEN
WE ANNOUNCE THE PROJECTS AND
NAME WHERE THOSE HOSPITALS ARE,
SO THE TEAMS CAN BRING THEM AND
WE CAN LEAN ON ARPANET H TO DO
THAT FOR SURE. WE WILL HAVE IT
AN ADVISORY BOARD OF SURGEONS IN
THE SEATS THE PROGRAM MANAGER
HAS ASSIGNED ON THAT BOARD. MANY
OF THOSE SEATS ARE RURAL AND
UNDERSERVED SURGEONS. BECAUSE
THEY WOULD BE THE END USERS, WE
WANT TO MAKE SURE THEY ARE
WILLING TO ADAPT TO TECHNOLOGIES
SO THERE WILL BE A KEY PLACE SO
IF YOU KNOW FOLKS THAT FIT THAT
BILL LOVE TO HEAR FROM THEM AS
WELL AS WE -- THOSE ON THE
ADVISORY BOARD.
>> ANNIE.
>> THANK YOU SO MUCH FOR BEING
HERE WITH US TODA TODAY. I HAVEE
PLEASURE AND PRIVILEGE OF
WORKING ON A CROSS STAKEHOLDER
GROUP THAT WORKED ON THE
AUTHORIZING LANGUAGE FOR HR AND
WE WORKED VERY HARD TO BE SURE
WE AVOIDED ANY TYPE OF WHAT WE
CALL DISEASE WARDS SO WE DIDN'T
MENTION ANY PARTICULAR DISEASES
BUT WE WORKED TO MAKE SURE THE
AUTHORIZING LANGUAGE USED
TERMINOLOGY THAT WOULD BE
DISEASE BASED AGNOSTIC TO USE
THE TOOLS TO DRIVE THE
INNOVATION. SO THE TERMS THAT WE
LANDED ON AND ENDED UNDERSTOOD
ENDING UP BEING IN THE OPERATING
LANGUAGE WERE TO LOOK FOR
PROJECTS THAT WOULD HAVE EFFECT
ON DISEASE BURDEN, UNMET PATIENT
NEED, DRIVE DECISIONS AND
POTENTIAL TO PREEMPT PROGRESSION
OF SERIOUS DISEASE. ONE OF THE
THINGS WE ARE INTERESTED IN
SINCE WE ARE HERE AT NCATS I'M
INTERESTED IN, I GUESS I'M
ALWAYS SURPRISED WHEN I SEE
DESCRIPTIONS OF ARPA H AND ONE
OF YOUR FIRST SLIDES DESCRIBE
ARPA H FOCUSED ON CANCER
ALZHEIMERS AND DIABETES. I
ABSOLUTELY UNDERSTAND AND
APPRECIATE YOUR DESCRIPTION HERE
TALKING EMPHASIS ON RARE DISEASE
BUT WITHIN THE AUTHORIZING
LANGUAGE WE WERE SUPER
INTENTIONAL TALKING ABOUT UNMET
NEEDS AND DISEASE BURDEN. SO I
GUESS I'M WONDERING CAN YOU TALK
A LITTLE BIT ABOUT AS YOU THINK
ABOUT THE PRIORITIZATION OF
RESOURCES AND RESOURCE
ALLOCATION, HAS THERE BEEN ANY
THOUGHT ABOUT HOW YOU ENSURE
INDEED WE ARE MAKING SURE WE
ACCELERATE BETTER HEALTH
OUTCOMES FOR EVERYONE AND ALL
THE MAJOR RESOURCES AREN'T GOING
TO COMMUNITIES WHO ARGUABLY ARE
RECEIVING SIGNIFICANT AMOUNT OF
RESOURCES FROM OTHER PROJECTS
ALREADY. AND MAKE THEM SAY WE
DO, I CARE DEEPLY, WE ALL CARE
DEEPLY ABOUT CANCER DIABETES AND
ALZHEIMER'S BUT THE RARE DISEASE
COMMUNITY IS NOT RECEIVING A
SIGNIFICANT PORTION OF RESOURCES
ALREADY AND WE VERY MUCH WOULD
LAKE TO SEA SIGNIFICANT PORTION
OF RESOURCES COME FROM ARP H BE
DEDICATED TO THOSE PROJECTS.
>> LET ME TRY TO UNPACK. SO I AM
FIRST THANK YOU FOR THE WORK
THAT YOU DID TO PUT THAT
AUTHORIZING LANGUAGE IN PLACE.
IT WAS ONE OF THE THINGS THAT I
MENTIONED EARLY ON THAT I WENT
ON THE HILL AND TALKED TO
MEMBERS OF THE CONGRESS ABOUT
THINGS UNCOMPROMISING ON. THE
THING I WAS INSISTENT WE WERE
UNCOMPROMISING ON WAS NOT A
SINGLE DOLLAR OF ARPA H DOLLAR
COULD BE LINKED TO SPECIFIC
DISEASE. WE HAVE TO HAVE TOTAL
FREEDOM OF WHAT TO FUND FROM THE
DISEASE PERSPECTIVE TO THE
TECHNOLOGY TO THE STATE, YOU
NAME IT, IT IS UP TO US TO
DECIDE. WE HAVE THAT AUTHORITY.
AND I WILL DEFEND THAT DURING
ALL OF MY TIME HERE AS DIRECTOR.
SO THERE IS NO LANGUAGE AROUND
ALZHEIMER'S OR CANCER OR
DIABETES. I KNOW THAT HAS
FLOATED OUT THERE. WE DON'T USE
THAT INTERNALLYLY. BUT TO SAY IF
WE HAVE A PROPOSAL OR PROGRAM
THAT PROGRAM MANAGER IDENTIFIES
A GAP THAT IS T NOT BEING
ADDRESSED WE DO THAT ON MARKET
ANALYSIS TO MAKE SURE THE
PRIVATE SECTOR IS NOT ALREADY
ADDRESSING IT INTERNAL AT NIH WE
HAVE A DECONFLICTING PROCESS TO
MAKE SURE THERE IS NOT PROJECTS
ALREADY FUNDED. WE DO THAT
DIGITALLY, 88,000 PROPOSAL AS
YEAR, WE LOOK THROUGH THOSE AND
WORK WITH PEOPLE AT NCATS OR THE
RELEVANT INSTITUTES TO ENSURE
OUR INVESTMENTS TRULY ARE UNIQUE
AND ONLY RELEVANT FOR ARPA H.
THEN ONCE WE DO ALL THOSE
THINGS, WE TRY TO MAP A
PORTFOLIO. WE DON'T SAY I CAN'T
TELL YOU WHAT WE ARE GOING TO
FUND THREE YEARS FROM NOW
BECAUSE I HAVEN'T MET THE
PROGRAM MANAGERS WHERE ET IN
MANY CASES SO WE DON'T HAVE A
PLAN THE NEXT TEN YEARS. BUT WE
DO HAVE THE PORTFOLIO MAP TO
MAKE SURE WHERE WE IDENTIFY
WHITE SPACE, I WILL GIVE COUPLE
OF EXAMPLES, WE HAVE WHITE SPACE
IN RARE DISEASE, WHITE SPACE IN
MATERNAL HEALTH AND WE HAVE
WHITE SPACE IN PEDIATRICS.
THESE ARE AREAS WE HAVE
IDENTIFIED AS A GAP THAT WE
THINK OUR INVESTMENT CAN MAKE AN
ASYMMETRICAL TO MANAGE. BUT THE
TRICK TO ARPA IS YOU NEED TO
CHAMPION TO COME IN AND BE THAT
PROGRAMS MANAGER IN PROJECTS
AROUND THAT AREA,S THAT MY POINT
AROUND THE FLY WHEEL. THE
PORTFOLIO THIS YEAR SHOULD LOOK
TOTALLY DIFFERENT THAN FIVE
YEARS FROM NOW. IF YOU LOOK AT
DARPA THAT IS EXACTLY THE CASE.
WHILE TODAY WE DON'T HAVE A RARE
DISEASE PROJECT I CERTAINLY HOPE
SEVERAL MONTHS FROM NOW WE WILL.
T THIS IS WHERE YOU BRING IN
YOUR COMMUNITY AND HELPING US
IDENTIFY THOSE CHAMPIONS WHO CAN
BE HERE FOR THREE YEARS OF A
TERM TO BE FULL CHAMPION EFFORT
THEN THEY WILL VACATE, LEAVE THE
SLOT OPEN FOR THE NEXT PERSON IS
WHAT IS IMPORTANT ABOUT THIS
AGENCY. I HOPE THAT MAKES SENSE
AND ANSWERS YOUR QUESTION.
>> IT DOES, HELPFUL TO
UNDERSTAND YOU IDENTIFYING THE
WHITE SPACES AND NOT FILLING ALL
THE FUNDING WITH WHATEVER THE
OTHER SPACE, SO THANK YOU VERY
MUCH.
>> I CAN MEET AFTERWARDS TO
SHARE OUT BUT IF YOU GO TO BROAD
AGENCY ANNOUNCEMENT, OPEN BAA
THE FOUR FOCUS AREAS THE HEALTH
SCIENCE SCALABLE SOLUTIONS UNDER
EACH IS ABOUT TEN EXAMPLES OF
AREAS WE THINK WE COULD HAVE A
OUTSIDE IMPACT. LIKE PEDIATRICS,
MATERNAL HEALTH, ADOLESCENT
MENTAL HEALTH. THESE ARE AREAS
WE THINK WE CAN MAKE IMPACT. SO
WE ARE FLAGGING TO THE COMMUNITY
HEY, WE NEED THOSE IDEAS BUT
AGAIN THOSE IDEAS COME FROM THE
OUTSIDE IN. IT IS -- IT DOESN'T
COME FROM US TOP DOWN.
>> RENEE, THANK YOU SO MUCH FOR
JOINING US TODAY. AND SPENDING
TIME WAS. I KNOW YOU HAVE BEEN
INCREDIBLY BUSY OVER THE LAST
FEW WEEKS, SEEN THE
ANNOUNCEMENTS SO APPRECIATE YOUR
TIME AND THANK YOU FOR THE
DISCUSSION.
>> THANK YOU ALL. APPRECIATE IT
APPRECIATE YOU JONI. BYE-BYE.
>> ALL RIGHT. TAKE CARE. SO WITH
THAT I THINK WE ARE TEN MINUTE BREAK.
I THINK WE ARE READY TO
RESUME. THANK YOU, EVERYONE,
WELCOME BACK. WE WILL START OUR
CONCEPT CLEARANCES NOW. SO LET'S
GO AHEAD AND MOVE FORWARD WITH
DR. DAN TAGLY WHO WILL TALK
ABOUT TISSUE CHIPS IN SPACE --
DR. TAGLE WHO WILL TALK TISSUE
CHIPS IN SPACE AND BEYOND.
>> GOOD AFTERNOON, THANK YOU,
JONI. I WILL BE TALKING THE
FIRST OF CONCEPT CLEARANCES FROM
NCATS, FIRST OFF WOULD BE TO
INTRODUCE OFFICE OF SPECIALTY
INITIATIVES AND TELL YOU MORE
ABOUT WHAT WE DO AS AN OFFICE.
SO OFFICE SPECIALIST SERVES
WITHIN NCATS ADDRESSES
TRANSLATIONAL PROBLEMS TO
CREATIVE SOLUTIONS BY INNOVATING
AND CREATING DISRUPTIVE
TECHNOLOGIES THROUGH NOVEL
PARTNERSHIPS THAT RESULTS IN
PARADIGM SHIFT. IN THE FIELD.
SO WE HAVE A NUMBER OF PROGRAMS
AND ACTIVITIES WITHIN OUR
OFFICE. AS YOU CAN SEE HERE IN
THIS SLIDE OUR TECHNOLOGIES SPAN
THE RANGE FROM FUNDAMENTAL
RESEARCH ALL THE WAY THROUGH
PRE-CLINICAL THROUGH REGULATORY
ACCEPTANCE. ALL THE PROGRAMS WE
DO PRIMARILY ADHERE TO THE NCATS
3-D PRINCIPLES OF DEVELOPING
TECHNOLOGY, DEMON INVESTIGATING
THE UTILITY OF THE TECHNOLOGY,
AND ULTIMATELY BEING ABLE TO
DISSEMINATE THE TECHNOLOGY FOR
PUBLIC USE. SO WE HAVE A NUMBER
OF DIFFERENT PROGRAMS, WITHIN
THE OFFICE HIGHLIGHTED IN BLUE
ARE TECHNOLOGIES THAT ARE
PRIMARILY AI DRIVEN, ARTIFICIAL
INTELLIGENCE OR INTELLIGENCE
MOSTLY AREA OF MANUFACTURING. Z
WHERE WILL CHEMISTRY, AUTOMATED
CHEMISTRY OR SIP THETIC BIOLOGY,
SHOWN IN PURPLE ARE DIAGNOSTIC
TECHNOLOGIES AS WELL AS
DETECTION TECHNOLOGIES SHOWN
THERE WOULD BE USING ELECTRONIC
NODE TECHNOLOGY AND WE HAVE ALSO
PRIVILEGED TO WORK IN ERA OF
COVID FOR RAPID ACCELERATION OF
DIAGNOSTICS. SHOWN IN PINK WOULD
BE NEW AREA IN TERMS OF QUANTUM
INFORMATION SCIENCES USING
QUANTUM COMPUTATIONAL AND
QUANTUM BIOSENSING TECHNOLOGIES.
IN YELLOW WOULD BE A PROGRAM OF
REGENERATIVE MEDICINE TISSUE
REPAIR AND WOUND HEALING. THEN
THE LIGHT BLUE WOULD BE NIH
COMMON FUND AREAS IN TERMS OF
EXOSOMES AND EXTRA CELLULAR RNA
AS WELL AS NEUROMODULATION. WE
ALSO HAVE PARTNERSHIPS WITH
ANOTHER AGENCY OUTSIDE OF NIH,
NSF LOOKING AT CYBER PHYSICAL
SYSTEMS AND ROBOTICS. THEN THE
LAST CATEGORY OF ACTIVITIES WE
HAVE IS SHOWN IN GREEN, MOSTLY
IN TERMS OF MICROFABRICATED
TISSUES, 3-D BIOPRINTING AND
TISSUE CHIPS. SO THE CONCEPT I
WILL BRING FORWARD TODAY WOULD
BE RELATED TO THE TISSUE CHIP
TECHNOLOGY THAT NCATS INVESTED
IN OVER THE PAST DECADE OR SO.
SO THE TITLE OF CONCEPT IS
TISSUE CHIPS IN SPACE 2.0.
CLDs AND BEYOND. I WILL GO
INTO DETAIL ABOUT THIS CONCEPT.
SO TISSUE CHIPS IN SPACE 2.0 IS
ESSENTIALLY A RENEWAL SO THIS
HAS BEEN PREVIOUSLY CONCEPT
CLEARED AND WE DID RUN A PROGRAM
OVER THE COURSE OF THE LAST FIVE
YEARS OR SO. SO INDUSTRY
RENEWAL OF TISSUE CHIP AND
PROGRAM WILL BE FOCUS ON
CONNECTIVITY AND INTEGRATION OF
MULTI--- MULTIPLE ORGAN SYSTEMS
AND TISSUE CHIPS THAT
APPROXIMATE MATE A HUMAN BODY ON
A CHIP SYSTEM. WE ALSO WANT TO
ESTABLISH THE USE OF IPS STEM
CELL DERIVED MATERIALS FROM
DIVERSE GROUP OF INDIVIDUALS,
THAT REPRESENTS SEX AND VARIOUS
ETHNICITIES AS WELL AS
DEVELOPMENTAL AGE. AND WE ARE
PRIMARILY LOOKING IN TERMS OF
INITIALLY LOOKING AT NASA AND
COMMERCIAL ASTRONAUTS PRIMARILY
TO SERVE AS THEIR OWN TISSUE
CHIP AVENUE TARS. AVATARS AND
BEING TRANSLATED FOR USE ON
EARTH TO HAVE PERSONALIZED CHIPS
FOR INDIVIDUALS. WE ALSO WANT TO
IN THIS RENEWAL INCORPORATE
ADVANCEMENTS THAT WE HAVE MADE
IN TERMS OF 3-D MICROFABRICATION
TISSUE CHIP TECHNOLOGY SUCH AS
BEING ABLE TO EXPAND CULTURE
LIFE BEYOND SIX MONTHS AS WELL
AS AUTOMATE AND MINIATURIZED
PLATFORMS. SO IN THIS
PARTICULAR RENEWAL, THE OFFICE
OF SPECIAL INITIATIVES PRIMARILY
WANTS TO IMPROVE DISEASE
OUTCOMES BY TRANSLATING
OBSERVATIONS FROM BIOMEDICAL
RESEARCH EXPERIMENTS IN SPACE
INTERVENTIONS THAT BENEFIT THE
HEALTH OF INDIVIDUALS AND PUBLIC
HERE ON EARTH. SO WHAT DOES THAT
LOOK LIKE? SO IN TERMS OF THE
BIOMEDICAL RESEARCH
OPPORTUNITIES, AS YOU CAN SEE
HERE BOTH IN TERMS OF LOW EARTH
ORBIT AND SPACE, THERE ARE
OPPORTUNITIES TO MODEL DISEASES
IN HUMAN CONDITIONS THAT MAY BE
DIFFICULT OR TAKE LONGER ON
EARTH. IN ONE PARTICULAR AREA
WOULD BE ON AGING. IT TAKES
OBVIOUSLY YEARS IF NOT DECADES
TO MODEL HUMAN AGING BUT UNDER
INFLUENCE OF MICROGRAVITY AS
TURNS OUT WE CAN DO SO IN A
MATTER OF WEEKS OR MONTHS.
THERE'S ALSO OPPORTUNITIES TO
BETTER UNDERSTAND EFFECT OF
COSMIC RADIATION, MICROGRAVITY,
AND PHYSICAL ISOLATION THAT
COULD DEVELOP EFFECTIVE
INTERVENTIONS AND COUNTER
MEASURES. OPPORTUNITIES ALSO
REST ON BIOMANUFACTURING,
ABILITY TO CRYSTALLIZE PROTEINS,
AS WELL AS BIOPRINT ORGAN
SYSTEMS UNDER MICROGRAVITY THAT
COULD BE HELPFUL FOR
REGENERATIVE MEDICINE. WE ARE
ALSO LOOKING AT OPPORTUNITIES
FOR TELEMETRY AND OTHER ROBOTIC
AND OTHER AUTONOMOUS SYSTEMS
THAT LEND ITSELF FOR UNDERSOURCE
OR UNDERSERVE AREAS IN TERMS OF
HEALTHCARE DELIVERY. THEN
LASTLY, WE ARE LOOKING TO ENABLE
POSSIBILITY OF CREATING LONG
SHELF LIFE PHARMACEUTICALS
AND/OR ON DEMAND PHARMACOLOGY
CAPABILITIES. SO WHAT HAS BEEN
DONE SO FAR? IN TERMS HOW HAS
THIS OPPORTUNITY BEEN TRANSLATED
FOR USE HERE ON EARTH? HERE ARE
JUST A FEW EXAMPLES WHERE NIH
HAVE MORE RECENTLY TRIED TO
CRYSTALLIZE KRAS, A ONCOPROTEIN
AS A WHOLE PROTEIN IT'S BEEN
DIFFICULT TO CRYSTALLIZE
ESPECIALLY WITH HYPERVARIABLE
REGION BEING INCORPORATED INTO
THE G DOMAIN. THAT HAS BEEN
POSSIBLE UNDER MICROGRAVITY. A
SECOND AREA THAT HAS COME OUT IN
TERMS OF BIOMEDICAL RESEARCH IN
SPACE IS TO IMPROVE PRODUCTION
PROCESSES FOR MONOCLONAL
ANTIBODY KETRUDO. IN THE PAST
DELIVERED BY IV INFUSION WHICH
TAKES SEVERAL HOURS AND REQUIRES
HOSPITALIZATION, BUT UNDER
MICROGRAVITY THIS IS UNIFORM
CRYSTALS IN SIMILAR THAN
TRANSLATED THAT PROCESS INTO
BEING ABLE TO MANUFACTURE
KEYTRUDA INTO HOMOGENOUS
CRYSTALS THAT HAVE BEEN ABLE SUB
Q INEX SOMEWHERE. MORE RELATED
DOWN TO EARTH IN TERMS OF HOW WE
HAVE ENABLED TISSUE CHIP
TECHNOLOGY TO INFORM BIOMEDICAL
RESEARCH OVER THE LAST FOUR OR
FIVE YEARS OR SO OF THE PROGRAM,
THAT'S BEEN RUNNING, WE HAVE
BEEN ABLE TO UNDERSTAND HOW
CARDIOVASCULAR DIP AND CHIP --
DECONDITIONING HAPPENS TO OUR
CARDIO MYSELFCYTES AND CHIPS,
SHOWN THAT SIX MONTHS OF
EXPOSURE UNDER MICROGRAVITY IS
EQUIVALENT TO TEN YEARS OF
BIOLOGICAL AGING SO YOU CAN
DEFINITELY STUDY THIS KIND OF
PROCESSES, PHYSIOLOGICAL
PROCESSES IN A MUCH MORE
ACCELERATED TIME LINE. WE HAVE
ALSO SHOWN DECREASE FORCE
CONTRACTILITY AS WELL AS SWITCH
FORCE NOT ONLY CARDIAC BUT
SKELETAL MUSCLE SO EFFECTIVELY
BEING ABLE TO USE THIS
INFORMATION TO STUDY SARCOPENIA
OR MUSCLE WASTING. AGAIN, THAT
IS SHOWN, MUSCLE LOSS IS
ACCELERATED FROM ONE PERCENT
NORMALLY HERE ON EARTH TO ABOUT
3% MUSCLE LOSS IN SPACE. WE
HAVE ALSO SHOWN BIOMARKER
CHANGES THAT ARE SIMILAR TO
THOSE OBSERVED FOR AGING OF THE
IMMUNE CELLS SUCH AS
IMMUNOSENESCENCE. LASTLY, WHAT
WE ARE TRYING TO DO IS MAKE
TECHNOLOGY THAT SUPPORTS AND
RUNS TISSUE CHIP TO BE AUTOMATED
AND TRANSFERABLE SO ALLOWING
ACCESS TO LOW INCOME AND MIDDLE
INCOME COUNTRIES. SO WHAT HAS
BEEN SHOWN AS I MENTION
MICROGRAVITY EXPOSURE INDUCES
AGING RELATED CHANGES. LIKE
SARCOPENIA, BONE DENSITY LOSS,
CARD YES VASCULAR
DECONDITIONING, GI DISTURBANCE,
CHANGES IN BLOOD CELLS,
DECLINING IMMUNI IMMUNITY. AND L
STONE INFLAMMATION. TISSUE CHIPS
AN SPACE PROGRAM WAS A
PARTNERSHIP WITH NCATS, NASA,
THE CENTER FOR ADVANCEMENT OF
SCIENCE IN SPACE AND
INTERNATIONAL SPACE STATION
NATIONAL LABORATORY. WE HAD TO
GO MODELING AGE RELATED DISEASE
UNDER MICROGRAVITY TO BE ABLE TO
TRANSLATE THE OPPORTUNITIES THAT
WE LEARNED FOR IMPROVEMENTS HERE
ON EFFORT. ONE OF THE THINGS
THAT CAME OUT OF THIS
PARTNERSHIP, NOVEL PARTNERSHIP
WAS WORKING WITH PAYLOAD
DEVELOPERS AN SPACE ENGINEERS
WITH ABILITY TO AUTOMATE AND
MINIATURIZE THE PLATFORM. SO
SHOWN HERE IS AN EXAMPLE HOW
THIS HAS HAPPENED. IN LOOKING AT
THIS AS A PROJECT BY JONATHAN
(INAUDIBLE) KIDNEY ON A CHIP SO
IN ORDER TO RUN 24 KIDNEY CHIPS
REQUIRES INCUBATOR SPACE ALONG
WITH THE CONTROLLERS, VALVES AND
TUBINGS, 48 CUBIC FEET THE SIZE
OF YOUR AVERAGE KITCHEN
REFRIGERATOR BUT CERTAINLY
WORKING WITH SPACEX AND NASA AND
BEING ABLE TO REALIZE THAT THE
PAYLOAD HAS TO BE REDUCED THE
SIZE OF A SHOE BOX. SO WE WORK
EFFECTIVELY WITH SPACE TANGO AND
BIOSERVE, SPACE IMPLEMENTATION
PARTNERS THAT BRING DOWN TO
PRACTICE AND BE ABLE TO
MINIATURIZE AND AUTOMATE
TECHNOLOGIES. SO THIS IS NOW IN
THE PROCESS OF TRYING TO TAKE
THIS TECHNOLOGICAL ADVANCEMENT
FOR USE HERE ON EARTH. SO THIS
SHOWS WE HAVE BEEN SUCCESSFUL IN
MINIMIZING THE TECHNOLOGY AND
AUTOMATING IT SO WE HAVE HAD A
HAD A TOTAL OF NINE LAUNCHES
EACH FIRST SET WAS TO SHOW
TECHNOLOGY CAN PERFORM UNDER
PRESSURE OF LAUNCH OPERATIONS
UNDER MICRO GRAVITY AND BRINGING
BACK THE SAMPLES HERE ON EARTH
FOR ANALYSIS. THE SECOND LAUNCH
WAS THEN TO LOOK AT THE
BIOMARKERS IDENTIFIED THAT WOULD
INDICATE ACCELERATED AGING AND
TO TEST COUNTER MEASURES OR
INTERVENTIONS THAT WILL SLOW
DOWN EFFECTS OF AGING SO IT CAN
BE MARKETED TO EVENTUAL USE HERE
ON EARTH. SO WHAT ARE WE LOOKING
FOR IN -- DURING RENEWAL, TISSUE
CHIPS 2.0. OBJECTIVES ARE
LOOKING AT DEPLOYMENT OF TISSUE
CHIPS AS GREATER MODELS OF
ACCELERATED AGING TO DEVELOP
INTERVENTIONS AND COUNTER
MEASURES TO MITIGATE AGING
EFFECTS HERE ON EARTH, DEPLOY
MULTI-ORGAN INTEGRATED PLATFORMS
THAT CLOSELY APPROXIMATE HUMAN
BODY IN A CHIP SYSTEM, TO BE
ABLE TO SEE THIS TISSUE CHIPS
WITH ORGAN SPECIFIC CELL TYPES
AND DIVERSE GROUPS OF PEOPLE FOR
PRECISION MEDICINE TO
INCORPORATE ADVANCEMENT IN
TISSUE CHIP TECHNOLOGY SUCH AS
CULTURE LIFE BEYOND SIX MONTHS
AND AUTOMATED AND MINIATURIZED
PLATFORMS TO BE ABLE TO ACCESS
-- ASSESS QUALITY AND INTEGRITY
OF STEM CELLS SUITABLE FOR IN
SPACE BIOMANUFACTURING INCLUDING
THE PRODUCTION OF THERAPEUTIC
EXOSOMES FOR REGENERATIVE
MEDICINE. AND THEN ENABLE 4D
BUYER PRINTING CAPABILITIES
UNDER MICROGRAVITY TO ALLOW FREE
FORM VOLUME METRIC PRINTING OF
COMPLEXION ORGAN CONSTRUCTS. SO
THE KEY AREAS OF EMPHASIS IN
THIS RENEWAL WOULD BE
PARTNERSHIP AGAIN WITH NASA AND
POSSIBLY WORKING OUT AGREEMENTS
WITH SPACE AGENCY AS WELL AS
CURRENT INTERNATIONAL SPACE
STATION NATIONAL LABORATORY AND
AS YOU KNOW THE CURRENT SPACE
STATION IS SCHEDULED TO BE
DECOMMISSIONED STARTING IN 2025.
SO THERE ARE NOW EFFORTS TO
BRING UP CLDs OR COMMERCIAL
LEGAL DESTINATIONS, THERE ARE
FOUR FROM ACTION SPACE, FROM
ORBITAL REEF WHICH IS FUNDED BY
BLUE ORIGIN AND ALSO LOOKING AT
LOCKHEED MARTIN AND NORTHRUP
GRUM MIRKSN. WE ARE WORKING WITH
INSTITUTES AN CENTERS AT THE NIH
TO PROVIDE FUNDING SUCH AS THE
NATIONAL INSTITUTE FOR AGING.
AND LOOKING AT ABILITY TO
POSITION TISSUE CHIPS TO BE
BIOMEDICAL EXPERIMENT OF CHOICE
BEYOND LOW EARTH ORBIT. THERE IS
NOW DISCUSSIONS IN TERMS OF
MISSION TO MOON AND MISSION TO
MARS. WE STILL DON'T KNOW WHAT
THE HEALTH HAZARDS THAT WE ARE
GOING TO BE EXPOSING THE
ASTRONAUTS AND EVENTUALLY SPACE
COLONIZERS, SO TALKING ABOUT
POSSIBILITIES OF USING TISSUE
CHIPS, TO BE PART OF THE MISSION
OF ARTEMIS III OR THE MISSION O
TO MARS WHERE TISSUE CHIPS WILL
BE POSITIONED TO ANTICIPATE WHAT
THE EXPOSURE HAZARDS WOULD BE
FOR FUTURE SPACE COLONIZERS. SO
WHY NCATS? CERTAINLY NCATS HAS
BEEN -- OCCURRED IN PREVIOUS
DISCUSSION, LEADER IN TISSUE
CHIP TECHNOLOGY HERE IN THE
UNITED STATES AS WELL AS
INTERNATIONALLY. WE ALSO SERVE
AS THE DR. JONI ROGERS SERVES AS
THE HHS AND NIH LIAISON TO NASA
IN TERMS OF INTERFACING AND
PARTNERING WITH THEM IN TERMS OF
BIOMEDICAL RESEARCH IN SPACE.
AND LOOKING AT WHAT ARE THE
EXPECTED OUTCOMES AND IMPACT OF
THIS NEW CONCEPT, WE SHOULD BE
IN A POSITION TO UNDERSTAND
HUMAN CELLULAR RESPONSES, SPACE
FLIGHT. AND BE ABLE TO
UNDERSTAND AND DEVELOP MODEL
SYSTEMS TO STUDY AGING EFFECTS.
PROVIDE KEY INSIGHTS O THE
CONTROL AND OPTIMIZATION OF STEM
CELL DIFFERENTIATION, SUCH AS
PLURIPOTENCY, MULTI-POTENCY. AS
WELL AS STEM CELLS TO
PROLIFERATE AND EXPAND UNDER
MICROGRAVITY, HOW WE CAN
ACCELERATE THE MATURATION
PROCESS UNDER MICROGRAVITY.
INCREASE USE OF EXOSOME BASED
THERAPIES FOR TISSUE REPAIR AND
WOUND HEALING. AND THEN BE ABLE
TO DEVELOP THERAPEUTICS AND
COUNTER MEASURES FOR
PHYSIOLOGICAL CHANGES.
ASSOCIATED WITH THE AGING
PROCESS. AND LASTLY BE ABLE TO
ENABLE TISSUE CHIP CORE
PRECISION MEDICINE TISSUE ON A
CHIP SYSTEM AND BUILD UP
CAPABILITIES FOR 4D BIOPRINTING
OR ORGAN PRODUCTION UNDER
MICROGRAVITY FOR USE IN
REGENERATIVE MEDICINE HERE ON
EARTH. IN TERMS OF WHAT WE
ANTICIPATE SUCCESS OF THIS
PROGRAM, WE WOULD LIKE AGAIN TO
REITERATE WE WILL BE -- TO MODEL
ACCELERATED AGING, 30% DIVERSE
POPULATIONS IN CHIPS TO DEVELOP
TISSUE CHIP AVATARS FOR USE IN
PRECISION MEDICINE. WE WOULD
LIKE TO ALSO LOOK AT DEVELOPMENT
OF COUNTER MEASURES AGAINST
AGING RADIATION EXPOSURE DUE TO
COSMIC RADIATION AN EVENTUALLY
TO HAVE A BETTER UNDERSTANDING
OF HUMAN BIOLOGY RELATES TO
CHRONIC EXPOSURE TO HAZARDS AND
RISKS ASSOCIATED WITH SPACE
FLIGHFLIGHT IN SUMMARY MAIN GOAF
TISSUE CHIPS 2.0 PUTTING FORTH
FOR COUNCIL FOR CONSIDERATION OF
RENEWAL IS TO BE ABLE TO IMPROVE
DISEASE OUTCOMES BY TRANSLATING
OBSERVATIONS FROM BIOMEDICAL
RESEARCH IN SPACE, INTO
INTERVENTIONS THAT BENEFIT
HEALTH OF INDIVIDUALS AND PUBLIC
ON EARTH. THIS WILL BE A CONCEPT
BUILT UPON ALREADY BY SUCCESSFUL
PARTNERSHIPS THAT NCATS HAD WITH
NASA, THE ISS AND PURSUING OTHER
PARTNERSHIPS. POTENTIALLY WITH
ARPA H AND COMMERCIAL VIDEO
DESTINATION AS WELL AS OTHER
INSTITUTES AND CENTERS AT THE
NIH. THIS INITIATIVE WILL
ADVANCE MULTIPLE OTHER RELATED
FIELDS RELATED TO TISSUE CHIPS
SUCH AS TISSUE ENGINEERING
MECHANIC KNOW BIOLOGY,
REGENERATIVE DISEASE MODELING
AND SPACE MEDICINE. SO IN THIS
LAST SLIDE JUST A FEW QUESTIONS
FOR COUNCIL FOR CONSIDERATION
AND COMMENTS IN TERMS OF WHAT
MIGHT IMPROVE THIS INITIATIVE
FROM PREVIOUS VERSION OR
PREVIOUS ITERATION. WHAT ARE
THE KEY POINTS TO CONSIDER THAT
I MAY HAVE MISSD IN THIS
RENEWAL. SPECIFIC QUESTIONS FOR
COUNCIL JUST TO PARAPHRASE
CAPTAIN JAMES KIRK FROM THE USS
ENTERPRISE, HOW CAN NCATS BEST
LEVERAGE TISSUE AVATARS TO GO
BOLDLY WHERE NO MAN HAS GONE
BEFORE. THANK YOU. HAPPY TO
ENTERTAIN ANY QUESTIONS.
>> THANK YOU, DAN. I BELIEVE WE
DO HAVE SOME DISCUSSION. I HAVE
MATTHIAS AND PAULA TO GO FIRST.
MATTHIAS, FIRST ON MY LIST, DO
YOU WANT TO START?
>> DAN, AS ALWAYS AS YOU HAVE
FOR THE LAST TWO DECADES, VERY
EXCITED TO SEE THIS CONTINUE AND
I WOULD SOLUTIONS DEVELOP IN
SPACE FOR US DOWN HERE ON EARTH.
THAT IS WELL ALIGNED WITH THE
NCATS MISSION BUT ALSO BE AWARE
SOLUTION DEVELOPED IN SPACE FOR
SPACE, THAT SHOULD BE PAID FOR
BY NASA. SO CONTINUE YOUR VERY
EFFECTIVE DISCUSSIONS, THIS CORE
FUNDING BECAUSE I DON'T THINK
NCATS FUNDING SHOULD TEACH US
HOW WE KEEP OUR PEOPLE ALIVE IN
SPACE. THAT IS A BIG ASK. I
SERVED ON VERY SUCCESSFUL MANY
THE PAST KEEP CORE FUNDINGS
MOVING FORWARD BECAUSE NASA
BENEFITED TREMENDOUSLY FROM ALL
THE INVESTMENTS N CATS HAS MADE
>> THANK Y THANK YOU. MATTHIAS.K
TO CONTINUE THAT PARTNERSHIP, AS
I POINTED OUT WE ARE ALSO
ENTERING A ERA IN SPACE MEDICINE
AND SPACE EXPLORATION BECAUSE
NASA HAS BEEN LOOKING IN TERMS
OF HOW TO COMMERCIALIZE SPACE.
THE REPLACEMENTS FOR
INTERNATIONAL SPACE STATION WILL
HAVE TO BE -- WILL BE PRIMARILY
PRIVATE FUNDING SO NAVIGATING IN
TERMS OF HOW WE PARTNER WITH
PRIVATE CORPORATIONS RIGHT NOW
SUCH AS ORBITAL REEF, AGAIN,
FUNDED BY JEFF BEZOS, BLUE
ORIGIN COMPANY, NOR THEREUPON
GRUMMIN LOCKHEED MARTIN, THOSE
ARE ALL NEW TERRITORYS WE ARE IN
THE PROCESS OF TRYING TO
DETERMINE WHAT THE BEST
PARTNERSHIP OPPORTUNITIES MAY
ARISE.
>> EXCELLENT KEY. GOOD LUCK.
WHAT WE CAN OFFER TO THOSE IS
THESE ARE KEY ASSETS WHO WILL
HELP TO DEVELOP PROGRAMS ALSO
FROM THE COMMERCIAL SIDE TO KEEP
PEOPLE SAFE. I THINK FROM THE
CONCEPT THE MAIN SUCCESS FROM MY
VANTAGE POINT WAS OF THE CURRENT
PROGRAMS THAT YOU REALLY FORCE
INVESTIGATORS TO DEVELOP
ULTIMATE SOLUTIONS FOR THEIR
PLATFORMS. THAT IS FOR ME ONE
OF THE MAIN DELIVERABLES AND
PROBABLY ONE OF THE CONSEQUENCES
WHY FDA NOW CONSIDERS THESE
PLATFORMS TO BE READY FOR
THERAPEUTIC USE. YOU SHOULD
ABSOLUTELY TAKE THA THA THAT.
>> THANK YOU FOR POINTING THAT
OUT. THAT IS PROBABLY IN SOME
WAYS THE MORE TANGIBLE EVIDENCE
OF THIS PRODUCTIVE PARTNERSHIP
WITH NASA IS THE CAPABILITY TO
DEMOCRATIZE TISSUE CHIP PLATFORM
BECAUSE EVEN NOW AS SHOWN ON THE
SLIDE, THE TECHNOLOGY IS VERY
PROMISING BUT THE SUPPORTING
INSTRUMENTATION CAN STILL BE
TECHNICALLY HARD AND WOULD
REQUIRE BIOMEDICAL ENGINEERS TO
RUN SUCH A SYSTEM. BUT WITH
AUTOMATION AND CERTAINLY WITH
MINIATURIZATION OF SMALLER FOOD
PRINT, I HARKEN BACK TO DAYS
WHEN I WAS WORKING EARLY DAYS OF
PCR WHEN YOU WOULD BE DUNKING
EPITOPES INTO WATER BATHS AND
YOU HAVE TO BE BABY SIT IT. THE
ASTRONAUTS DON'T HAVE THE TIME
TO BABY SIT EXPERIMENTS IN SPACE
SO AUTOMATION IS A KEY FEATURE
IN TERMS OF STANDING BIOMEDICAL
RESEARCH IN SPACE, SO I THINK
THE AUTOMATION AND
MINIATURIZATION CAPABILITIES
THAT WE HAVE LEARNED WILL THEN
LEND ITSELF TO ALMOST LIKE A PCR
TYPE MACHINE INSTRUMENTATION FOR
TISSUE CHIPS WHERE YOU CAN PLUG
IN YOUR CHIPS, WALK AWAY, AND
COME BACK AND BE ABLE TO LOOK AT
THE DATA AS IT IS ALSO BEING
COLLECTED. SO I THINK THAT THAT
WOULD CERTAINLY LEND ITSELF FOR
WIDESPREAD ADOPTION NOT ONLY
WILL IT MAKE IT EASIER TO
OPERATE BUT IT ALSO MAKE IT
SUFFICIENT TO REQUIRE SUCH
TECHNOLOGICAL CAPABILITIES.
WHICH LEADS TO NEXT CASE HOW DO
YOU (INAUDIBLE) KELLY TWINS ONE
UP THERE AND ONE DOWN HERE WHICH
REALLY PROVIDED LOT OF HARD
EVIDENCE OF THE ACCELERATED
AGENING SPAC SPACE.
>> THAT WAS A KEY INSIGHT IN
TERMS OF HOW SCOTT (INAUDIBLE)
CHANGED PHYSIOLOGICALLY BUT IN
SOME WAYS SURPRISINGLY TOO THAT
MOST OF THE FUZZ OWE LOGICAL
CHANGES REVERTED BACK
UNFORTUNATELY FOR HIM REVERTED
BACK TO NORMAL WHEN HE RETURNED
BACK TO EARTH BUT THAT GIVES US
OPPORTUNITY TO THEN WHAT ARE THE
MOLECULAR SIGNATURES THAT
CHANGES WHEN YOU ARE EXPOSED TO
MICROGRAVITY THAT INSTITUTE THIS
-- ALL THE PHYSIOLOGICAL CHANGES
UNDER MICROGRAVITY RELATED TO
AGING. AND THEN LOOKING BACK AT
THE SIGNATURES THEN RETURNING TO
EARTH, SO THAT WE CAN BETTER
UNDERSTAND HOW WE CAN REVERSE OR
SLOW DOWN EFFECTS OF AGING. WE
ONLY HAVE ONE IDENTICAL TWINS
WHO ARE ASTRONAUTS SO THAT IS
OUR GOAL TO CREATE TISSUE CHIP
AVATARS INSTEAD OF HAVING
GENETIC TWINS WE WILL HAVE
TISSUE CHIPS WITH EVERYONE WHO
MIGHT BE CANDIDATE TO GO OUT THE
SPACE TO GO ALONG TISSUE CHIP
AVATAR TO IN DEPTH PROBING OF
PHYSIOLOGICAL CHANGES THAT MIGHT
BE HAPPENING TO THEM. SO THAT
GIVES US ALSO THE CAPABILITY
HERE ON EARTH TO CREATE TISSUE
CHIP AVATARS FOR DIAGNOSTICS AS
WELL AS PRECISION MEDICINE
APPLICATION.
>> I THIS I THAT IS MY FINAL
COMMENT, BENCHMARKING THOSE
SYSTEMS WILL AGAINST IN VIVO
STATE OBVIOUSLY YOU HAVE BEEN
DOING THROUGHOUT YOUR WILL BE
MORE RELEVANT AND WILL BE
PARTICULARLY RELEVANT TO MAKE
SURE THAT THE INSIDE OF THE
ACCELERATED AGING POLICIES WE
HAVE A FEW IDEAS WHAT
ACCELERATES BUT I DON'T THINK WE
HAVE ALL ANSWERS YET, IS
BECOMING AVAILABLE SO YOU KNOW
WHICH PROCESSES ACCELERATED OF
IT AND WHICH ONES ARE MAINTAINED
TO THE STATE WE HAVE FOUND HERE.
SO TECHNOLOGIES HAVE RAPIDLY
MATURED TO DO THAT, THIS
GENOMICS DATA STRUCTURE AND FROM
VERY MINDED ELEMENTS BUT
BENCHMARKING AGAINST THE
INDIVIDUALS WHERE YOU HAVE THE
IPC MODEST TISSUE SAFE, CREATIVE
SOLUTIONS NOW ALSO BECOMING
AVAILABLE MIGHT BE ONE ASPECT TO
CONSIDER.
>> THANK YOU. CERTAINLY WILL BE
CAREFUL TO HAVE THOSE
BENCHMARKS.
>> AND WE ARE A RESULT OVER TIME
SO WE MOVE US -- A LITTLE OVER
TIME SO MOVE TO NEXT QUESTION.
>> I'M INCREDIBLY ENTHUSIASTIC
ABOUT THIS PROGRAM. I DO BELIEVE
OUR NEXT GENERATION IS GOING TO
BE SPENDING LOTS OF TIME IN
SPACE AND THAT IS JUST GOING TO
BE YET ANOTHER TRANSLATIONAL
ISSUE FOR NCATS TO DEAL WITH AND
TO ACCELERATE NOT ONLY WHAT IS
GOING TO BE THE BENEFITS HERE ON
EARTH BUT ALSO HOW YOU KEEP
PEOPLE SAFE IN SPACE. I THINK IF
YOU CAN DO SOME OF THESE THINGS
IN SPACE WITH ASTRONAUTS WHO
AREN'T BIOLOGISTS YOU CAN DO
THEM IN RURAL AMERICA AND IN
UNDERSERVED PLACES AS WELL. SO I
AM VERY ENTHUSIASTIC ABOUT THE
PARTNERSHIP BECAUSE NO ONE
FUNDING STREAM WILL BE ABLE TO
DO THIS ALONE, IT WILL TAKE
MULTIPLE FEDERAL PRIVATE AND
ACADEMIC AND MANY, MANY OTHERS
TO TRY TO MAKE THIS WORK AND TO
MAKE THIS GO. I DO THINK THAT
HELPING PERHAPS THE ACADEMICS
AND HELPING THE OTHERS IN HOW TO
DESIGN CHIPS, AND NCATS IS A
WORLD LEADER IN THIS AND WE
DISCUSSED A LITTLE BIT HOW CHIPS
THAT WORK ON EARTH MAY NOT WORK
IN REMOTE EARTH ORBIT OR OTHER
ATMOSPHERES SO THAT HELPING THE
COMMUNITY THAT DOES HAVE DEEP
EXPERTISE IN DIFFERENT ASPECTS
OF AGING AND BONE LOSS AND
MUSCLE AND HOW YOU DEAL WITH
THESE CHIPS AND HOW PROGRAMS IN
ADDITION TO WORK WITH THAT
PUBLIC PRIVATE ACADEMIC
PARTNERSHIP SO WE CAN DESIGN
THESE CHIPS THAT CAN BE USED NOT
ONLY IN SPACE BUT ULTIMATELY IN
OTHER PLACES. ONCE AGAIN, VERY,
VERY THRILLED ABOUT THIS
PROGRAM. KEEP UP THE GREAT WORK.
>> THANK YOU, DR. SHIREMAN.
>> THANK YOU SO MUCH FOR THAT
INPUT AND NOW I WILL TURN IT
OVER TO YOU.
>> IF THERE IS NO FURTHER
DISCUSSION ON THIS CONCEPT,
MOTION TO APPROVE THE TISSUE
CHIPS CONCEPT? MOVED. SECOND?
>> HE COULD IS.
>> ALL IN FAVOR?
>> AYE.
>> ANY OPPOSED? ANY ABSTENTIONS
WITH THAT, THE TISSUE CHIP
CONCEPT IS APPROVED. NEXT.
>> THANK YOU. ALL RIGHT NEXT WE
HAVE A PROGRAMMATIC UPDATE FROM
THE DIVISION OF CLINICAL
INNOVATIONS WITH DR. MIKE
KURILLA. SO MIKE, OVER TO YOU.
>> ALL RIGHT. IT IS A PLEASURE.
I'M GOING TO GIVE YOU A -- AM I
ADVANCING SLIDES I? THE CO-BOAR.
OKAY. -- KEYBOARD. I'M GOING TO
GIVE YOU A WHIRLWIND TOUR OF THE
CTSA PROGRAM, VERY HIGH-LEVEL,
VERY SELECTIVE. I WISH WE HAD
THE OPPORTUNITY TO RUN THROUGH
LOT MORE DETAIL BUT WE ONLY HAVE
SO MUCH TIME SO WE WILL TRY TO
GET THROUGH THIS. I SHOWED THIS
EARLIER, CTSA PROGRAM. THIS IS
REALLY THE FOCUS. I COULD
PROBABLY PICK ANY ONE OF THESE
AND SPEND THE REST OF THE
AFTERNOON TALKING ABOUT THE
ACTIVITIES SO I WILL TRY TO DO
AN OVERVIEW. JONI SHOWED THIS
EARLIER, THIS IS JUST TO SAY
THAT UNFORTUNATELY WITH REGARD
TO BUDGET WE ARE LIVING, WE --
THE BEST CASE SCENARIO TO END UP
NEXT YEAR WITH WHAT WE HAD THIS
PAST YEAR. SO THAT IS WHERE WE
ARE. THE FY 23 CTSA PROGRAM H
CURRENT HUBS AS YOU CAN SEE WE
HAVE A TOTAL OF 64, THERE WERE
16 UM 1 AWARDS INDICATED BY THE
RED DIAMONDS. SO IT IS A PRETTY
-- WE FEEL PRETTY SUCCESSFUL
PRETTY SATISFIED WITH THE
SUCCESS OF WHAT THIS PAST YEAR
HAS BEEN IN TERMS OF NEW FOAs.
I WANT TO SHARE, NOT TOO MUCH
DETAIL BUT THERE WAS SOME
CONCERN EXPRESSED BY THE
COMMUNITY INITIALLY AS WELL AS
CONGRESS THAT THE CHANGES WERE
GOING TO REDUCE THE SUPPORT TO
HUBS OVERALL. SO WE DID A
CAREFUL ANALYSIS, ALL 16 FOR THE
ENTIRE YEAR, WE WERE TOLD
EVERYONE HAD TO BE AT LEAST 95%.
AS YOU CAN SEE ONLY 2 OUT OF 16
SHOWED ANY DECLINE. ONE WAS
PRETTY INSIGNIFICANT, ONE ONLY A
DECLINE OF 3%. BUT OVERALL THE
AVERAGE INCREASE FOR THE
CTSAs, THERE WAS A 14%
INCREASES IN TOTAL AMOUNT GOING
IN TERMS OF THE UM 1s RELATIVE
TO THE UL-1s AT THE SAME HUBS
THE PREVIOUS YEAR. SO YOU CAN
SEE IT WAS A RATHER SIZABLE
INCREASE OF NEARLY $14 MILLION
FOR THOSE 16 INSTITUTIONS, I
HAVE TO GIVE CREDIT TO ANDY
KELLY AN TEAM IN TERMS OF THE
MODELING AND FORECASTING THEY
DID, THEY WERE VERY MUCH ON
TARGET. THIS IS WHAT WE
ANTICIPATED AND THIS IS HOW
THINGS TURNED OUT. WITH REGARD
TO THE K PROGRAM, 12 OF THE 16
DID RECEIVE Ks. NOT ALL OF
THEM DID, THERE WERE A FEW THAT
DIDN'T DO AS WELL IN REVIEW,
WHICH IS NOT A BAD THING BECAUSE
IF WE HAD BEEN UNDER THE OLD
U-54, THAT POOR K APPLICATION
MIGHT HAVE TANKED THE OVERALL
AND WOULD HAVE LOST THE U
PORTION AS WELL. BUT WE ALSO
HAVE IN INSTANCES WHERE
INSTITUTIONS WISH TO DEFER AWARD
OF THEIR K, TO LATER IN THE NEXT
YEAR IN ORDER TO BETTER ALIGN IT
TO THE ACADEMIC CALENDAR, THAT
HAS BEEN A RECOMMENDATION BY
SEVERAL, WE HAD SEEN THAT
SEVERAL TIMES IN TERMS OF
PREVIOUS RFIs THAT SOME
INSTITUTIONS ACTUALLY WANTED A
SEPARATION OF THE Us AND THE
Ks SO THEY COULD HAVE BETTER
ALIGNMENT TO THEIR ACADEMIC
CALENDAR SO THEY HAVE TAKEN
ADVANTAGE OF THAT. AS YOU CAN
SEE OUR MODELING ANDY'S CREW WAS
REALLY RIGHT ON, YOU CAN SEE
THERE IS A TOTAL INCREASE IN
AMOUNT OF SUPPORT GOING TO THE
Ks. WE DID FIND IN OUR
ANALYSIS OF THE Ks THAT THERE
WAS QUITE BIT OF, I WILL BE NICE
AND SAY THERE IS QUITE A BIT OF
CREATIVITY WHO PEOPLE INSTITUTED
THEIR K PROGRAMS WHICH CREATED
DIVERSITY. THIS CREATES SOME
DOWNSTREAM PROBLEMS IN TERMS OF
REPORTING. , COMPLIANCE
FOLLOW-UP FROM THE GRANTS
MANAGEMENT SIDE, SO INJECTING A
LITTLE UNIFORMITY, WE KNEW THERE
WERE GOING TO BE MAYBE A FEW NOT
EVERYONE COME OUT ON TOP BUT
OVERALL AS YOU CAN SEE IN TERMS
OF SLOT NUMBER, THE OVERALL SLOT
NUMBERS ACTUALLY WENT UP AN ONLY
1 OF 12 SAW DECLINE ONLY IN ONE
SLOT SO OVERALL THE MODELING HAS
HELD UP PRETTY WELL AND WE ANDY
AN CREW NAILED IT AS FAR AS I'M
CONCERNED. STAFF UPDATES, WE
HAVE HAD A NUMBER OF NEW PEOPLE
IN PARTICULAR, YOU WILL SEE AT
THE FAR RIGHT ROBIN WAGNER IS
THE DIRECTOR OF OUR OFFICE OF
PROGRAM EVALUATION ANALYSIS AND
RECORDING. WE CALL IT OPARE.
AND WE HOPE TO BE ABLE TO HAVE
BETTER COORDINATION AND
HARMONIZATION ACROSS THE
DIVISION IN TERMS OF ACTIVITIES
GOING ON IN THE HUB, ACROSS THE
CTSA PROGRAMS. CONGRATULATIONS
ARE IN ORDER. JONI SHOWED THIS
EARLIER, I WANT TO SAY THAT N 3C
I WON'T TALK TOO MUCH ABOUT IT I
HAVE TALKED ABOUT IT EXTENSIVELY
IN THE PAST. THIS AWARD
HIGHLIGHTS THE CONTRIBUTIONS
THEY HAVE MADE TO THAT I WILL
HIGHLIGHT WERE INSTITUTED BY
QUESTIONS COMING FROM THE WHITE
HOUSE. AT ONE POINT THERE WAS
CONCERN INITIALLY DURING THE
PAXLOVID ROLL OUT, THERE WAS A
LOT OF QUESTIONS AND N 3C
STEPPED UP AND ADDRESSED
PAXLOVID REBOUND. WHEN WE FIRST
ASKED ABOUT THIS, IT WAS SAID
NO, YES NOT ABLE TO TELL ABOUT
PAXLOVID REBOUND IN N 3C BECAUSE
IT IS NOT DIAGNOSED THAT WAY.
BUT THE ISSUE WE RECOGNIZED WAS
THAT IT WASN'T PAXLOVID REBOUND
PER SE BUT THERE WAS THAT
REBOUND HAVING SOME CLINICAL
IMPLICATIONS SO N 3C ADDRESSED
THE FACT THAT INDIVIDUALS WHO
RECEIVED PAXLOVID WERE NOT
HAVING ANY SUBSTANTIAL INCREASES
IN TOUCHES WITH THE HEALTHCARE
SYSTEM AFTER PAXLOVID,
SUGGESTING IT WAS NOT A MAJOR
CONCERN. THAT ALLAYED A LOT OF
CONCERNS AND FEARS THAT PEOPLE
HAD THAT WE WERE SITTING ON A
TICKING TIME BOMB INITIALLY. AS
TURNS OUTS THAT'S BORNE OUT
WELL, WITH REBOUND MAY OCCUR BUT
NOT LEADING TO REPEAT
HOSPITALIZATIONS INCREASED
MORTALITY OR THINGS LIKE THAT.
THE OTHER ONE, LAST ONE WAS ONE
OF THE LAST THINGS AJIJ BEFORE
HE LEFT THE WHITE HOUSE ASKED US
ABOUT PAXLOVID UTILIZATION AND N
3C WAS ABLE TO DO A NICE
DETAILED ANALYSIS LOOKING AT
FROM ELIGIBILITY REQUIREMENTS
WHO WAS ELIGIBLE FOR PAXLOVID,
WHO WAS GETTING IT AND HOW IT
VARIED ACROSS INSTITUTION, IT
WAS VERY INTERESTING IN TERMS OF
RESULTS BECAUSE IT WAS FAR
UNDERUTILIZATION OF PAXLOVID
THAT I THINK PEOPLE APPRECIATED
AN ESTIMATING THE TENS OF
THOUSANDS OF LIVES THAT COULD
HAVE BEEN SAVED IF PAXLOVID HAS
BEEN ADMINISTERED BY THE
ELIGIBILITY REQUIREMENTS BUT IN
SOME CASES, THERE WAS ZERO
UTILIZATION BY INSTITUTIONS FOR
PAXLOVID SO WE JUST FOUND THERE
WAS A LOT OF UNEVENNESS ACROSS
THE COUNTRY. REMAINS TO BE SEEN
HOW WE CAN ADDRESS THAT IN THE
FUTURE. WE SEE THIS EVERY YEAR,
THE NUMBER OF NEW MEMBERS TO THE
NATIONAL ACADEMY OF SCIENCES
THAT HAVE AFFILIATIONS WITH THE
CTSA. CONTINUES TO BE QUITE
STRONG AND I THINK THIS REALLY
IS INDICATOR OF THE QUALITY OF
THE CTSA LEADERSHIP AND QUALITY
OF THE TYPES OF INDIVIDUALS THAT
ARE BEING SELECTED AND PROMOTED
WITHIN THE CTSA PROGRAM. PEOPLE
MAY RECALL THAT ONE OF OUR CTSA
PIs, BECK CAN YOU JACKSON
PASSED AWAY AND ACTS INSTITUTED
THE DISTINGUISHED EDUCATOR
RESEARCH DISTINGUISHED EDUCATOR
AWARD, RUN BY CYNTHIA MORRIS, A
PI, CTSA PI AT OREGON AND THEN
STEVE DUBINETT FROM UCLA MOVED
UP TO BECOME DEAN. SO HE'S
TURNED THE REIGNS OVER TO ARLENE
BROWN AT UCLA. I ALWAYS LIKE TO
SEE THESE, CTSA PEOPLE WHO ARE
BRILLIANT NEW INVESTIGATORS AN
BREAK THROUGH IS ALWAYS NICE TO
BE RECOGNIZED FOR THESE TYPES OF
SPECIAL AWARDS. THEN FOR THE
YOUNGER CROWD, THERE IS A NUMBER
OF THESE NATIONAL 40 UNDER 40
HONOREES. YOU CAN SEE FORMER TL
1, FORMER KL 2 SCHOLAR WHICH
INDICATES THE CTS ASAs ARE
DOING A GOOD JOB IDENTIFYING
PEOPLE WHO GO ON TO BE
RECOGNIZED BEYOND THEIR
INSTITUTION, FOR THE SIGNIFICANT
CONTRIBUTIONS. SO THERE'S A LOT
MORE BUT I DON'T HAVE ENOUGH
TIME TO GO INTO THAT. WANTED TO
JUST HIGHLIGHT THE RO 3 AS I
SAID BEFORE WE ARE DIPPING OUR
TOE IN THE WATER IN TERMS OF
MOVING TOWARDS AN R PROGRAM. AND
THE -- THIS SMALL GRANT PROGRAM
IS REALLY GEARED TOWARDS THE K
SCHOLARS TRYING TO HELP THEM
ALONG THE GRANTS PATHWAY AND I
THINK THIS IS A VERY BROAD GROUP
HERE, ONE OF THE ONES THAT
REALLY I FIND VERY INTERESTING
WHICH I'M GLAD TO SEE SOME FOCUS
ON, IF YOU LOOK AT THE FIRST ONE
BY ELIZABETH WOLF, IT'S LOOKING
AT BARRIERS TO DEIMPLEMENTATION
WE PUT EFFORT INTO
IMPLEMENTATION SCIENCE, DON'T AS
MUCH FOCUS ON DEIMPLEMENTATION
WHEN WE KNOW SOMETHING DOESN'T
WORK, HOW DO WE GET PEOPLE TO
STOP DOING THE STUFF THAT
DOESN'T WORK? IT IS ACTUALLY NOT
A TRIVIAL TASK. SO THAT IS WHAT
HER FOCUS WILL BE. AND HEN THE
NEXT ONE I LIKE INTERESTINGLY
BECAUSE IT DEALS WITH ADVANCED
CHRONIC EYE DISEASE TREATMENT.
BESIDES THE FACT I'M GETTING TO
THE POINT WHERE I NEED TWO SETS
OF READING GLASSES NOW WHICH IS
VERY FRUSTRATING BUT MORE
IMPORTANTLY I THINK TO ME IT
UNDERSCORE IT IS FACT THAT WE
PUT EFFORT INTO TALKING ABOUT
NIH AND PUT A LOT OF EFFORT
TALKING ABOUT SAVING LIVES.
NOBODY REALLY DIES OF EYE
DISEASE PER SE UNLESS YOU HAVE
OCULAR ROTE KNOW BLAST TOE MANY
OR SOMETHING LIKE THAT OR OCULAR
MELANOMA. BUT IN GENERAL I THINK
WE DON'T ALWAYS FOCUS AS MUCH ON
QUALITY OF LIFE ISSUES.S.
MAINTAINING VISION, HEARING,
MOBILITY, ARE VERY, VERY
IMPORTANT AND MAY NOT KILL
PEOPLE BUT I THINK IMPACTING THE
QUALITY OF PEOPLE'S LIVES IS
EQUALLY IMPORTANT, IF NOT MORE
IMPORTANT THAN SIMPLY EXTENDING
LIFE. WE ONLY HAD ONE CCIA AWARD
THIS YEAR THAT WAS GIVEN. THIS
IS INTEGRATION AN
INTEROPERABILITY OF COMPLEX DATA
AND TISSUE THE HUMAN BRAIN. I
THINK THIS IS A CONSEQUENCE OF
COMING OFF COVID, THERE WAS A
REAL SORT OF INTERRUPTION IN
THESE SORTS OF TYPES OF
PARTICULARLY CCIAs WERE
INVOLVED IN COLLABORATION AMONG
MULTIPLE CTSAs. I'M HOPING AS
WE ARE IN OUR WIND DOWN FROM THE
COVID PHASE, THAT COLLABORATIONS
ACROSS INSTITUTIONS WILL
INCREASE BUT I THINK THIS IS --
THIS KIND OF WORK I THINK IS
VERY INDICATIVE WHERE I SEE A
LOT OF FOCUS OF THE CTSAs
MOVING FORWARD BECAUSE THIS IS
REALLY LOOKING AT THE
INTEGRATION OF MULTIPLE TYPES OF
DATA TYPES, IF YOU LOOK HERE
LOOKING INCLUDING EEGs, MRI,
GENOMICS BLOOD SAMPLES
ELECTRONIC HEALTH RECORDS
BRINGING TOGETHER THE MASSIVE
AMOUNTS OF DATA IN A PRODUCTIVE
MANNER THAT REALLY IDENTIFIES
FUTURE AREAS OF INVESTIGATION I
THINK IS A KEY FOCUS ON WHAT WE
IMMEDIATE TO BE THINKING ABOUT
WHEN WE TALK
I LIKE TO POINT OUT TO PEOPLE
THAT IT'S NOT SIMPLY A MATTER OF
DOING THINGS FASTER.
WE SHOULD THINK ABOUT DOING
THINGS DIFFERENTLY, AND NEW
THINGS WE HAVEN'T THOUGHT TO DO
BEFORE.
AND THIS TYPE OF ANALYSIS I
THINK IS GOING TO PROMOTE THAT.
ONE OF THE THINGS I'M GOING
TO -- I'VE BEEN PUTTING MORE
ATTENTION INTO RECENTLY,
PARTICULARLY WITH REGARD TO THE
CCIAs, IS WHAT HAPPENS WITH
THESE.
I TELL YOU ABOUT MAKING THE
AWARDS BUT THERE'S NOT A
FOLLOW-UP.
UNLESS IT'S BROUGHT TO MY
ATTENTION BY THE INVESTIGATORS
WHICH IT WAS, OR BY MY STAFF OR
IN SOME CASES THE MEDIA.
THIS IS ONE SUCH CCIA THAT HAS
RATHER HERCULEAN TITLE OF HAR
HARNESSING THE POWER USING
SOCIAL DETERMINANTS OF HEALTH,
FRAILTY, FUNCTIONAL STATUS TO
IDENTIFY AT-RISK PATIENTS AND
IMPROVE RISK ADJUSTMENT, AWARDED
IN 2018.
SO IT'S JUST ABOUT COMING TO THE
END, YOU CAN SEE THE
COLLABORATING INSTITUTION,
UNIVERSITY OF TEXAS AT AUSTIN,
TEXAS HEALTH SCIENCE CENTER,
HOUSTON, UNIVERSITY OF
PITTSBURGH, YOU SEE THREE
PUBLICATIONS, THAT'S THREE OF
37, STILL COUNTING.
THIS IS A VERY, VERY IMPRESSIVE
PIECE OF WORK THAT REALLY
BROUGHT TOGETHER I THINK AND
ADVANCING A CONCEPT THAT I THINK
NEEDS MORE ATTENTION, THE
CONCEPT OF FRAILTY.
MEDICINE DOES A LOT OF THINGS
BECAUSE IT'S EASY TO DO.
AGE IS A VERY EASY THING TO DO.
IF YOU'RE OVER 65 YOU'RE AT
SOMETHING.
IF YOU'RE UNDER 65 YOU'RE NOT AT
RISK.
AGE IS ACTUALLY A PRETTY POOR
STRATIFIER.
IT DOESN'T REALLY WORK THAT
WELL.
WHAT WE REALLY ARE TALKING ABOUT
WHEN WE TALK ABOUT AGE IS WE'RE
FOCUSED ON FRAILTY AND BEING
ABLE TO QUANTIFY AND QUANTITATE
THAT.
IT'S INTERESTING BECAUSE WHEN
PEOPLE FIRST STARTED TO CONSIDER
THE POSSIBILITY OF ANTI-AGING
INTERVENTIONS, THE FDA SAID WE
CAN'T DEAL WITH THAT.
AGING IS NOT A DISEASE.
YOU CAN'T HAVE A DRUG AGAINST
SOMETHING THAT'S NOT A DISEASE.
CONCEPT OF FRAILTY WAS THOUGHT
TO BE AN APPROPRIATE SURROGATE,
UNDERSTANDING FRAILTY AND HOW
WOE MEASURE THAT AND HOW THAT
PREDICTS RISK FOR OTHER THINGS I
THINK IS CRITICAL.
THAT'S WHAT A LOT OF THIS CCIA
HAS REALLY BEEN INVOLVED WITH,
REALLY LOOKING AT A LOT OF
FACTORS AS WELL AS SOCIAL
DETERMINANTS OF HEALTH WHICH IS
CLEARLY ANOTHER UNDER UTILIZED
ACTIVITY OR AREA WE NEED TO
EXPLOIT MUCH MORE.
ONE OF THE OTHER THINGS WE DID
IN 23 WAS RECOMPETED THE TIN,
TIN 2.0.
I WANTED TO HIGHLIGHT EFFORTS
OVER THE LAST 7 YEARS, IN
PARTICULAR, PARTICULARLY WITH
COVID, THE TIN REALLY SHOWED
WHAT IT'S CAPABLE OF BUT I THINK
WE DON'T NECESSARILY RECOGNIZE
THE TOTALITY OF WHAT THE TIN HAS
BEEN ABLE TO DO.
ONE OF THE IMPRESSIVE NUMBERS
HERE FOR ME IS THAT 20 NIH
I.C.s HAVE ENGAGED THE TIN,
THAT'S IMPRESSIVE IN TERMS OF
CROSS-NIH RECOGNIZING
FUNCTIONALITY AND CAPABILITY AND
FUNCTIONALITY OF THE TIN.
THERE WERE 400 REQUESTS FOR
SUPPORT, 95% OF CTSAs
SUBMITTED PROPOSALS TO THE TIN
SO IT'S GOTTEN QUITE A BIT OF
ATTENTION AND UTILIZATION AND
RECOGNITION.
WE CAN THINK ABOUT RECRUITMENT,
ONE SMALL COMPONENT FROM
AWARENESS THROUGH TO RETURN OF
VALUE.
ONE OF THE INTERESTING ASPECTS
I'VE BEEN INVOLVED WITH CITY,
PRE-COVID HAD DISCUSSIONS OF
EUROPEAN POSITION OF RETURN OF
INFORMATION, RETURN OF RESULTS
OF CLINICAL TRIALS, AND THAT WAS
GOING TO HAVE AN IMPACT ON THE
U.S. AND COVID HIT, SO THAT
DISCUSSION STOPPED.
BUT UNDERSTANDING HOW WE DO THAT
AND RETURN VALUE BACK TO THE
PATIENTS WHO HAVE BEEN IN THE
CLINICAL TRIALS I THINK IS VERY
IMPORTANT.
THE RIC HAS REALLY PIONEERED A
NUMBER OF FEATURES, REDCap
CLEARLY IS ONE OF THE WELL
INTERNATIONALLY RESPONSIVE AND
UTILIZED COMPONENTS BUT THE
COMMUNITY ENGAGEMENT STUDIOS HAS
BEEN WILDLY SUCCESSFUL, AND
DURING COVID WAS HIGHLY, HIGHLY
UTILIZED IN TERMS OF ASSISTING
WITH THOSE.
I WANTED TO HIGHLIGHT ONE RIC
SUCCESS STORY.
THIS WAS THE TARGET TRIAL.
THIS IS ONE, FORMER NIAMS TOLD
ME THE RIC WAS RESPONSIBLE FOR
SALVAGING THIS TRIAL BECAUSE
AFTER THEY HAD INITIATED THE
TRIAL ON THEIR OWN, AFTER THREE
YEARS HAD 30 PEOPLE, AND THEY
WERE SHOOTING FOR 300.
THE PROGRAM OFFICER OVER AT
NIAMS WAS LOOKING AT A 30-YEAR
TRIAL AND KNEW THAT WASN'T GOING
TO WORK AND ACTUALLY REACHED OUT
AND DIRECTLY CONTACTED THE RIC
ABOUT THIS.
THE RIC WAS ABLE TO DO A NUMBER
OF REALLY -- REALLY SALVAGED
THIS WITH REFINEMENT OF
RECRUITMENT MATERIALS, IT WAS
PASSED OUT TO PATIENTS.
THE ORIGINAL RECRUITMENT
MATERIAL THAT THEY PROVIDED THE
PATIENTS ACTUALLY SPENT HALF OF
THE BROCHURE OUTLINING THE
CLEVER ACRONYM THEY HAD COME UP
WITH FOR CALLING IT THE TARGET
TRIAL.
WHICH I DON'T THINK PATIENTS
WERE MUCH INTERESTED IN.
THEY PRODUCED A STUDY APP THAT
WAS VERY EASY FOR A HEALTH CARE
PROVIDER TO PUT ON THEIR PHONE
AND PUT IN A FEW PARTICULARS
ABOUT THEIR PARTICULAR PATIENT
THEY WERE SEARING AND DETERMINED
IMMEDIATELY WHETHER OR NOT THAT
PATIENT WAS ELIGIBLE.
THE OUTCOME WAS THEY WERE ABLE
TO COMPLETE THE ENROLLMENT
WITHIN THE ORIGINAL TIME FRAME
SO THE RIC CAME THROUGH.
THE THE GROUP HAS COME BACK TO
THE RIC WITH A NEW GRANT
SUBMISSION, HELP WE'RE PLEASED
WITH THIS, AND THE END RESULT
THIS WAS A SALVAGED TRIAL THAT
WAS DETERMINING WHETHER OR NOT
FOR PEOPLE WHO WERE NOT DOING
WELL WITH THEIR RHEUMATOID
ARTHRITIS TREATMENT ON TWO DRUGS
WHETHER ADDING THIRD DRUG OR TNF
INHIBITOR WAS AN ADVANTAGE, SO
THIS IS HAS HAD A DIRECT IMPACT
ON PATIENT CLINICAL GUIDANCE.
SO THE TIN 2.0, WE HAVE TWO, ONE
AT VANDERBILT, ONE AT HOPKINS.
THE RISK RISK SUCCESSFULLY
COMPETED WITH THE VANDERBILT
MEHARRY GOING ON, AWARDED THIS
PAST SUMMER.
I THINK WE'RE PLANNING ON OUR
FIRST IN-PERSON MEETING SOMETIME
IN LATE NOVEMBER, I BELIEVE,
BEING SCHEDULED.
BUT THESE ARE SOME OF THE THEMES
THAT TIN 2.0 IS WORKING ON,
EXPANDING PARTICIPATION,
OUTREACH MORE TO HBCUs,
MINORITY-SERVING INSTITUTIONS,
RURAL ORGANIZATIONS TRYING TO
GET THEM MORE FOCUSED.
REALLY LOOKING AT
INCLUSION/EXCLUSION CRITERIA,
THIS IS ONE OF THE BIG ISSUES,
IT'S ONE OF THESE DIRTY LITTLE
SECRETS NIH DOESN'T WANT TO TALK
ABOUT.
SOMETIMES INCLUSION/EXCLUSION
CRITERIA PRODUCES LIMITATIONS,
WE DON'T RECOGNIZE FULLY GOING
IN HOW RESTRICTIVE THAT CAN BE.
WE HAD AN RFI OUT THIS YEAR
REGARDING DECENTRALIZED TRIAL
SUPPORT, AN ENORMOUS AMOUNT OF
DESIRE AND INTEREST IN NOT ONLY
BY THE ACADEMIC COMMUNITY BUT
PHARMACEUTICAL COMMUNITY FOR
UTILIZATION OF DECENTRALIZED
TRIALS, ABILITY TO DO LOW TOUCH
OR NO TOUCH TRIALS.
WE'VE HAD CONTACT WITH THE FDA
VERY MUCH INTERESTED IN THE
ABILITY TO VALIDATE USE OF MORE
CLINICAL ENDPOINTS FROM DIGITAL
ANT MOBILE TECHNOLOGIES BECAUSE
THEY FEEL THIS WOULD GREATLY
IMPROVE ENROLLMENT AND DIVERSITY
RECRUITMENT AFTER ITS ACROSS.
THERE'S A LOT OF INTEREST IN
THIS AREA MOVING FORWARD.
AND I THINK SOME OF THE THINGS
WE'VE SEEN REALLY OFFER
OPPORTUNITIES FOR HOW WE CAN
REALLY ADVANCE THIS AREA.
CONSENT AND REGULATORY BURDEN
REDUCTION IS SOMETHING WE CAN'T
BE GIVING PEOPLE 50-PAGE
INFORMED CONSENTS.
IT JUST DOESN'T WORK.
AND THEN, YOU KNOW, OBVIOUSLY
SOME OF THE STANDARD THINGS,
TESTING TOOLS FOR TRAINING,
DISSEMINATION, CONTINUING WITH
THE TIN 1.0, WHICH WAS
MULTI-CENTER TRIAL SUPPORT
ACROSS TIN SERVICE ACTIVITIES
AND A LOT OF WORKING MORE
CLOSELY WITH CTSAs TO BETTER
UTILIZE THOSE.
YOU KNOW, I HIGHLIGHTED -- THIS
WAS HIGHLIGHTED IN THE
SUPPLEMENTS, ADMINISTRATIVE
SUPPLEMENTS THIS MORNING.
THIS IS RECOGNITION BEYOND
NCATS, PLACES ACROSS NIH THAT
CAME TO NCATS TO PROVIDE MONEY
FOR ACTIVITIES THAT CAN BE
UTILIZED THROUGH THE CTSA
PROGRAM, INCLUDING "HEAL."
AND TRAINING PROGRAMS AS WELL AS
BIOETHICS, AS WELL AS A FEW
OTHER TOPICS.
SO, I WANT TO TALK ABOUT SOME OF
OUR WHAT I CONSIDER OUR MOST
VALUABLE PLAYERS.
SO, I HIGHLIGHT A COUPLE KL2
SCHOLARS, H.
THIS IS AN INTERESTING ACTIVITY
IN ARKANSAS.
ARC
ARKANSAS IS UNIQUE.
MOST PEOPLE ARE PROBABLY NOT AS
OLD AS I AM, SO, DURING --
SHORTLY AFTER WORLD WAR II WHEN
THE U.S. WAS TESTING ABOVE AT
ATMOSPHERIC NUCLEAR BOMB TESTING
WE BLEW UP THINGS IN THE PACIFIC
AND RELOCATED PEOPLE FROM THE
MARSHALL ISLANDS WHERE WE BLEW
UP SOME ATOLLS IN THAT REGION.
AND WE RELOCATED THEM TO
NORTHWEST ARKANSAS.
AND SO THE UNIVERSITY OF
ARKANSAS CTSA HAS HAD AN
OUTREACH AND A LONGSTANDING
COMMUNITY ENGAGEMENT WITH THAT
MARSHALESE POPULATION, REACHING
OUT TO THE REPUBLIC OF THE
MARSHALL ISLANDS THROUGH THE
CHURCH CONNECTION, FAITH-BASED
ORGANIZATIONS, IN NORTHWEST
ARKANSAS AND THERE, TO LOOK AT
SOME -- WHAT AMOUNTS TO SOME
STRIKING HEALTH DISPARITIES THEY
HAVE SOME OF THE HIGHEST AGE
ADJUSTED TYPE 2 DIABETES
MELLITUS IN THE WORLD, OFFERING
POTENTIAL INSIGHT INTO DISEASE
ETIOLOGY AND PATHOGENESIS.
AND SO SHE'S DONE A GREAT JOB IN
TERMS OF MOVING THAT EFFORT
FORWARD.
GREGORY PECK, WHO IS AT THE
RUTGERS CTSA, I'M PARTICULARLY
FOND OF THIS BECAUSE WE DON'T
ALWAYS SEE SOMEBODY ACTUALLY
DOING PREVENTIVE CARE.
I THINK IT'S SOMETHING THAT CAME
OUT IN MANY OF THE STAKEHOLDER
ENGAGEMENTS, THAT REALLY NEED TO
FOCUS MORE IN PREVENTING DISEASE
RATHER THAN JUST THINKING ABOUT
TREATING IT AFTER OCCURS.
HE'S PUT QUITE A BIT OF EFFORTS
INTO PREVENTIVE CARE TO DECREASE
RISK FACTORS FOR CHRONIC DISEASE
BEFORE THE NEED FOR
HOSPITAL-BASED TREATMENT
ACTUALLY ARISING.
I LIKE TO TALK ABOUT FORMER K
SCHOLARS, CAROLYN IS BECOMING A
ROCK STAR.
SHE IS THE LEAD INVESTIGATOR ON
THE COVID-OUT TRIAL LEADING TO
POSITIVE RESULTS FOR METFORMIN
FOR ACUTE COVID AND ALSO
DISPLAYED INTERESTINGLY ENOUGH A
42% REDUCTION IN THE DEVELOPMENT
OF LONG COVID FOLLOWING ACUTE --
FOLLOWING TREATMENT FOR ACUTE
COVID, WHICH IS IMPRESSIVE, AND
ONE OF THE REASONS THAT ACTIV 6
IS FOLLOWING ON TO SEE IF WE CAN
REPEAT THERE'S, THERE'S QUITE A
BIT OF INTEREST GIVEN THE
COMPLETE LACK OF ANY SPECIFIC
INTERVENTIONS TO ADDRESS LONG
COVID AT THIS POINT.
SO, SHE'S JUST REALLY TAKEN OFF
AND DONE QUITE IMPRESSIVE WORK
IN THIS AREA.
SO FOR TL1s, ANDREW RAINEY,
TALKED ABOUT THIS BEFORE, BUT I
THINK THE UTILIZATION OF
WASTEWATER AS A PUBLIC HEALTH
MEASURE I THINK PRE-COVID PEOPLE
WOULD HAVE RECOMMENDED A PSYCH
EVALUATION IF YOU REQUESTED THIS
BUT IT'S CLEARLY SHOWN, THE
ABILITY TO USE THIS IN A
COMPREHENSIVE MANNER REQUIRES
THE ABILITY TO UNDERSTAND HOW WE
GO ABOUT DOING THIS AND
NORMALIZATION FACTORS FOR
EPIHERE, ANALYZING DATASETS OF
182 COMMUNITIES ACROSS MULTIPLE
STATES.
IT IS -- THIS IS REALLY GETTING
AT THE BEGINNINGS OF HOW WE CAN
REALLY BEGIN TO UTILIZE THIS
INFORMATION FOR VERY EFFECTIVE
PUBLIC HEALTH GUIDANCE.
YOU'LL HEAR MORE ABOUT ANOTHER
PUBLIC HEALTH PROJECT RELATED TO
THIS LATER ON.
SALLY EAGLETON, LOOKING AT
IMPROVING SCREENING AND
REFERRALS FOR FEDERAL NUTRITION
PROGRAMS PARTICIPATION, USING
ELECTRONIC HEALTH RECORDS.
KEEP IN MIND THAT THIS IS THE
WIC -- WE HAD THE PROBLEM WITH
THE BABY FORMULA, WIC IS ONE OF
THE MAIN DRIVERS OF THAT.
SO THIS REALLY HAS A BIG IMPACT.
BUT THE ABILITY TO INTEGRATE A
LOT OF ELECTRONIC HEALTH RECORD
DATA INTO THIS ANALYSIS I THINK
IS SOMETHING THAT'S BEEN SORELY
LACKING AND GREATLY NEEDED.
SO WHERE DO THESE GO ON TO?
WELL, THIS INDIVIDUAL QUITE
IMPRESSED ME, ZAK DYER IS A
FORMER TL1 TRAINEE FROM LAST
YEAR, WENT ON TO GET A
SUBSEQUENT T32 PRE-DOCTORAL
FELLOWSHIP FROM NIMHD, FOCUSED
ON A -- LOOKING AT A LARGE
NUMBER, 40 DATA ELEMENTS,
MEASURES SOCIAL DETERMINANTS OF
HEALTH TO PROVIDE SUPPORT FOR A
STRUCTURAL RACISM EFFECT INDEX
WHICH PEOPLE ARE ACTUALLY QUITE
INTERESTED IN.
BUT HE'S AN MD/PHD STUDENT AT
THE UNIVERSITY OF MASSACHUSETTS.
BUT HE'S TEMPORARILY ON LEAVE
BECAUSE AS A STUDENT HE'S BECOME
THE ACTING DIRECTOR OF THE
WORCESTER DEPARTMENT OF PUBLIC
HEALTH, SO I THINK IT'S PRETTY
IMPRESSIVE WHAT HE'S DOING SO
FAR, AND HOPEFULLY HE WILL
COMPLETE HIS Ph.D. AND HIS
WORK WITH A LOT OF PEOPLE
LOOKING FORWARD IT.
WITH REGARD TO OUR DIVERSITY AND
REENTRY SUPPLEMENT AWARDEES WE
HAD SEVEN THIS PAST YEAR, NICE
RANGE FROM GEOGRAPHICALLY ACROSS
THE COUNTRY.
AND JUST TO HIGHLIGHT TWO, LAIKA
IS ASSISTANT PROFESSOR OF
PSYCHIATRY AT UCSD, AND SHE IS
LEVERAGING MACHINE LEARNING,
PREDICTORS FOR EMERGENCE OF
SUBSTANCE ABUSE WHICH IS
PROBABLY A FRUITFUL AREA OF
INVESTIGATION.
AND THEN CATRELL, ASSISTANT
PROFESSOR OF EPIDEMIOLOGY,
UNIVERSITY OF KENTUCKY, SLEEP IN
APPALACHIAN CHILDREN.
WHEN I VISITED KENTUCKY THEY
TOLD ME EASTERN KENTUCKY, THEY
ARE ON THE EDGE OF, IS VERY MUCH
LIKE WEST VIRGINIA.
SO IT'S A SIGNIFICANT POPULATION
WHEN YOU THINK ABOUT WHAT GOES
ON IN WEST VIRGINIA, EASTERN
KENTUCKY IS SUFFERING VERY
SIMILAR.
AND SO THERE ARE A LOT OF
ISSUES, BODY MASS INDEX,
SECONDHAND SMOKE.
THIS IS REALLY IMPACTING THE
PEDIATRIC COHORT QUITE
SIGNIFICANTLY THERE.
AND SO WHERE ARE THEY NOW?
DMITRY IS NOW ASSISTANT
PROFESSOR OF MEDICINE, ASSOCIATE
DIRECTOR OF HEALTH EQUITY,
UNIVERSITY OF COLORADO.
HER FOCUS IS ON PERIPHERAL
ARTERY DISEASE IN AFRICAN
AMERICANS.
SO, I'LL RUN THROUGH A COUPLE
LITTLE HIGHLIGHTS HERE.
NEWS HIGHLIGHTS.
WE'LL BE SEEING MORE AND MORE OF
THIS.
A.I. APPLICATIONS, WHAT WE'RE
LOOKING AT IS INFORMATION
EVERYWHERE, SO THIS IS WHAT MAY
BE CONSIDERED A SLIGHTLY OBSCURE
STUDY.
I DON'T KNOW THAT PEOPLE ARE
REGULARLY READING WHATEVER THIS
JOURNAL IS.
BUT WHAT THEY WERE ABLE TO DO,
THIS GROUP AT VANDERBILT, WAS TO
ACTUALLY TAKE ROUTINE CHEST
CTs, AS YOU CAN SEE HERE, AND
THEY WERE ABLE TO ACTUALLY
EVALUATE IT, CHEST CTs,
SUBCUTANEOUS ADIPOSE TISSUE,
MUSCLE MASS, BONE, IF YOU HAVE
TO DISTINGUISH BETWEEN MEN AND
WOMEN OBVIOUSLY.
BUT THEY WERE ABLE TO DERIVE
OVERALL BODY COMPOSITION FROM
THESE.
WHICH IS PRETTY IMPRESSIVE.
SO WHAT IT MEANS IS IF YOU'RE
LOOKING AT A CHEST CT FOR THE
PURPOSE OF WHAT'S GOING ON IN
THE CHEST, YOU'RE LEAVING A LOT
OF INFORMATION ON THE TABLE.
THERE'S A LOT OF DIGITAL
INFORMATION THAT THE A.I. WAS
ABLE TO REALLY PULL OUT.
WHAT THEY HAVE SUBSEQUENTLY GONE
ON TO DO WITH THIS, WHICH IS
WHAT I WANTED TO HIGHLIGHT, WAS
THEY HAVE TAKEN THIS AND USING
LOW DOSE TOMOGRAPHY DATA, CHEST
CTs, THAT THE NCI HAS FUNDED
OVER DECADES FOR LOW-DOSE LUNG
SCREENINGS FOR PEOPLE AT HIGH
RISK FOR LUNG CANCER, HAVE TENS
OF THOUSANDS OF THESE CHEST
CTs THEY HAVE BEEN TRACKING
FOR YEARS AND YEARS AND YEARS.
THEY HAVE BEEN ABLE TO SHOW BY
STRATIFYING IN TERMS OF MUSCLE
MASS AND ADIPOSE TISSUE,
RELATIVE ADIPOSITY AND
SARCOPENIA, IF YOU STRATIFY YOU
CAN SHOW YOU SEE A CORRELATION
IN TERMS OF NOT ONLY WITH
MORTALITY FROM LUNG CANCER BUT
MORTALITY FROM CARDIOVASCULAR
DISEASE AND ALL-CAUSE MORTALITY.
SO, ONE SIMPLE LOW DOSE
SCREENING CAN ACTUALLY PROVIDE
SOME VERY INSIGHTFUL VALUE
BEYOND JUST LUNG CANCER
DETECTION AND LUNG CANCER
FOLLOW-UP HERE.
I THINK THIS HIGHLIGHTS TO ME
THAT IN A LOT OF OUR IMAGING
MODALITIES THERE'S A LOT OF
INFORMATION THAT WE'RE NOT
RECOGNIZING AS YET, AND
ARTIFICIAL INTELLIGENCE IS
PROBABLY ONE WAY WE'LL BE ABLE
TO BE TEASE SOME OF THESE
RELATIONSHIPS OUT AND DO USEFUL
THINGS IN THE FUTURE.
I THINK ANOTHER AREA RIPE FOR
A.I., THIS IS A PAPER THAT CAME
OUT OF THE UCLA CTSA.
SO LOOKING AT CONSENT DOCUMENT
TRANSLATION.
WHAT YOU CAN SEE IS THAT IF YOU
COMPARE INDUSTRY TRIALS, THIS IS
ALL THE TRIALS DONE AT UCLA, IF
YOU COMPARE INDUSTRY TRIALS WITH
NON-INDUSTRY, WHICH IS LARGELY
ALMOST EXCLUSIVELY NIH-FUNDED
TRIALS, WHAT YOU FIND IS THAT
THE NIH-FUNDED TRIALS ARE ONLY
PROVIDING TRANSLATED INFORMED
CONSENT AT HALF THE RATE
INDUSTRY IS.
NOW, WHEN WE THINK OF
TRANSLATION WE MAY BE THINKING
IT'S MOSTLY SPANISH TRANSLATION
BUT IN SOUTHERN CALIFORNIA
YOU'RE PROBABLY TALKING ABOUT
UPWARDS OF 70 DIFFERENT
LANGUAGES THAT ARE SPOKEN, IF
YOU WANT TO GET IN ALL THOSE
COMMUNITIES.
SO WHEN THEY LOOKED INTO WHY
THIS WAS OCCURRING, IT WAS CLEAR
THAT THE ABILITY TO DO THAT
TRANSLATION, THE FUNDING WAS
JUST NOT AVAILABLE IN THE NIH
GRANTS TO SUPPORT THOSE
TRANSLATIONS.
INDUSTRY, IF THEY WANTED TO GET
TO THOSE PEOPLE, IF THEY WANT TO
HIT ENROLLMENT TARGETS WILL DO
WHAT IT TAKES WERE WILLING TO
PAY FOR IT BUT FOR NIH TRIALS
THAT I WAS -- THIS WAS AN AREA
OF LIMITATION.
A.I. IS BEING USED FOR
TRANSLATION, NOT AS SIMPLE AS
TELLING A.I. TO TRANSLATE BUT
THIS IS ONE AREA WHERE WE REALLY
SHOULD BE ABLE TO EXPLOIT A.I.
TO REALLY BE ABLE -- AND THIS
WOULD BE A SIMPLE
STRAIGHTFORWARD ENHANCEMENT TO
RECRUITMENT EFFORTS.
SO I MENTIONED THE WASTE WATER
BEFORE.
THIS CAUGHT MY EYE.
THIS IS OUT OF DAVIS.
IT HAS THE GREAT TITLE WHICH
CATCHES ME, ANYTIME YOU USE
BAYESIAN I'M INTERESTED, BECAUSE
USUALLY WHEN SOMEBODY SAYS
BAYESIAN EVERYBODY ELSE SAYS AS
THEY ARE CROSSING ONIONS IN
FRONT OF THEM, THIS IS A
BAYESIAN SEQUENTIAL APPROACH TO
MONITOR COVID VARIANTS FROM
WASTEWATER.
THEY ACTUALLY DID AN ANALYSIS
THAT COULD PROVIDE STATISTICAL
METHOD TO QUANTITATE THRESHOLD
LEVELS FOR ACTUALLY ESTIMATING
COMMUNITY TRANSMISSION,
COMMUNITY TRANSMISSION RATES OF
COVID FROM THE WASTEWATER DATA.
THIS IS SOMETHING THAT IS --
THIS IS REALLY NEWS YOU CAN USE
IMMEDIATELY.
WHAT'S QUITE IMPRESSIVE THOUGH
ABOUT THIS WAS THAT WHEN YOU
LOOK AT WHAT HAD TO BE BROUGHT
TOGETHER SO THESE WERE
DEPARTMENTS OF PUBLIC HEALTH
SCIENCES, MATHEMATICS, AND CIVIL
AND ENVIRONMENTAL ENGINEERING
GROUPS IN ORDER TO PULL THIS
OFF.
I THINK THIS IS, TO ME,
INDICATIVE OF WHERE THE FUTURE
WITH BIG DATA IS REALLY COMING
FROM THAT LOTS OF DIFFERENT
SPECIALTIES, LOTS OF EXPERTISE
AND DIVERSE SECTORS OF
SCIENTIFIC AND TECHNICAL
EXPERIENCE REALLY HAVE TO COME
TOGETHER IN SUCH A WAY TO BE
ABLE TO UTILIZE WHAT IS VERY
COMPLEX SCIENTIFIC AND MEDICAL
INFORMATION IN A PRODUCTIVE WAY
TO REALLY MAKE SOME -- COME UP
WITH VERY SPECIFIC GUIDANCE AND
RECOMMENDATIONS FOR PUBLIC
HEALTH.
AND SO I THINK THIS IS WHAT THE
CTSA ARE FACILE AT AND EMPHASIS
NEEDS TO BE EMPHASIZED FOR THE
FUTURE.
A FEW OTHERS, YOU KNOW,
SMARTPHONES, NEARLY EVERYONE
PROBABLY HAS ONE IN THEIR
POCKET, RIGHT IN FRONT OF THEM
RIGHT NOW.
AND SO I WAS REALLY IMPRESSED.
THIS IS OUT OF THE UCSD, THEY
HAVE A GEM PROGRAM WHICH IS AN
INTERESTING COMMUNITY WHERE THE
CTSA SPONSORS ABILITY TO BRING
TOGETHER MEMBERS OF SCHOOL OF
ENGINEERING FROM SCHOOL OF
MEDICINE AND THE SCHOOL OF
MEDICINE TALKS ABOUT THEIR
PROBLEMS OR ISSUES, AND THE
SCHOOL OF ENGINEERING COMES UP
WITH VERY INNOVATIVE SOLUTIONS.
AND SO WE MAY THINK BLOOD
PRESSURE TESTING IS PRETTY
ROUTINE, YOU CAN BUY SOMETHING
IN THE STORE, AT THE PHARMACY,
TO TAKE YOUR OWN BLOOD PRESSURE
BUT WE KNOW IT'S NOT TRIVIAL.
YOU HAVE TO BE CAREFUL ABOUT
TAKING THE MEASUREMENTS, GET THE
RIGHT CUFF SIZE, DO IT
CORRECTLY, ALL OF THIS.
BUT THIS WAS ACTUALLY A LITTLE
ATTACHMENT COSTING LESS THAN A
DOLLAR TO MANUFACTURE, THAT YOU
PLUG INTO A SMARTPHONE AND CAN
TAKE A BLOOD PRESSURE.
THIS REALLY THINK GOES A LONG
WAY IN TERMS OF DEMOCRATIZING
MONITORING OF BLOOD PRESSURE.
ONE CAN THINK NOT ONLY ABOUT
TAKING CARE OF PATIENTS IN THE
FUTURE BUT THE ABILITY TO DO
CERTAIN TYPES OF CLINICAL
TRIALS IN A VERY DECENTRALIZED
MANNER BECAUSE YOU DON'T HAVE TO
RELY ON DIFFERENT WAY TO RELY ON
OBTAINING INFORMATION SUCH AS
BLOOD PRESSURE.
I THINK WE'RE JUST AT THE
BEGINNING.
WE'RE ON THE CUSP OF WHERE
LITTLE TINY ATTACHMENTS
RELATIVELY CHEAP THAT CANNING
PLUGGED INTO SMARTPHONES WILL
EXPAND THE CAPABILITY, REALLY
PUTTING WHAT AMOUNTS TO A
CLINICAL LABORATORY IN THE PALM
OF YOUR HAND AND WILL REALLY
GIVE PEOPLE THE OPPORTUNITY TO
CONSIDER CERTAIN TYPES OF
CLINICAL STUDIES AND CLINICAL
TRIALS THAT WOULD HAVE BEEN
UNTENABLE PRIOR.
AND THEN THIS LAST ONE THAT I'LL
FOCUS ON, AGAIN, I THINK THIS IS
BRINGING TOGETHER DISPARATE
DATASETS, THIS IS FROM
UNIVERSITY OF NORTH CAROLINA.
SO, EVERYBODY I THINK WOULD WALK
AROUND AND CONSIDER THAT
POLLUTION IS NOT A GOOD THING.
AND BUT IT'S BEEN QUANTITATIVE
SO PARTICULATE MANNER
2.5-MICRON, WHAT THEY HAVE BEEN
ABLE TO SHOW, AGAIN I THINK THIS
IS WHERE BRINGING TOGETHER
DISPARATE DATASETS BUT THEY WERE
ABLE TO SHOW THAT THE PM2.5
EXPOSURE, THAT CREATED MORE
CARDIOVASCULAR MORTALITY AND
ENDED UP WITH MORE DIAGNOSTIC
TESTING PERFORMED, MORE
PROCEDURES AS A RESULT OF THAT.
AGAIN, THIS IS WHERE WE GET INTO
BIG DATA SO OVER 20,000 EHR
RECORDS WITH HEART FAILURE
SPECIFICALLY WERE EVALUATED.
AND THIS CAN ACTUALLY PROVIDE
SOME RECOMMENDATIONS AND
GUIDANCE, AND FOR BOTH
HEALTHCARE PROVIDERS AND
INDIVIDUALS, WITH PARTICULAR
CARDIOVASCULAR CONDITIONS.
AND I THINK THIS IS JUST --
WE'RE JUST AT THE BEGINNING OF
BEING ABLE TO GET THINGS LIKE
THIS.
I'M GOING TO FINISH UP WITH A
COUPLE EXAMPLES OF SOMEWHAT I
THINK ARE CTSA IMPACTS.
SO, I PRESENTED THIS, THIS IS A
SLIDE I PRESENTED MANY YEARS
AGO, PRE-COVID, SO SORT OF LIKE
BACK IN THE DARK AGES AS FAR AS
I'M CONCERNED.
HARD TO REMEMBER EXACTLY WHEN.
BUT BACK IN AROUND AT THE END OF
2007, EARLY 2008, THERE WAS A
CTSA PILOT AWARD.
THIS IS AT CORNELL.
WAS GIVEN FOR A PRE-CLINICAL
MODEL USING ULTRA SMALL
T UMOR-TARGETING CORE-SHELL
NANOPARTICLES.
THIS IS IT ON THE RIGHT, A
FAIRLY MALLEABLE PLATFORM THAT
ALLOWS YOU TO ATTACH A NUMBER OF
DIFFERENT THINGS.
YOU CAN HAVE A TARGETING
MOLECULE HERE, AND THEN CAN YOU
HAVE A RADIO LABEL, STICK A DRUG
ON, A LOT OF THINGS.
YOU WOULD INJECT THESE.
THEY SHOWED QUITE A BIT OF
RELATIVE SAFETY.
THERE WAS JUST EFFICIENT URINARY
EXCRETION FOR THEM.
AND IN 2011 THEY GOT AN IND FOR
A MULTIMODAL PLATFORM.
2014 THEY GOT IT FROM AN OPTICAL
PARTICLE PROBE.
THIS HAS GONE ON THOUGH, I
DIDN'T CREATE THIS.
THIS IS FROM A MICHELLE
BRADBURY, BUT THIS IS THE
ORIGINAL AWARD THAT WAS GIVEN
LATE 2007, EARLY 2008.
THEY GOT A SUBSEQUENT SEED GRANT
FROM THE CTSA AWARD, BUT WHAT
YOU CAN SEE IS THIS HAS GONE ON,
THERE'S BEEN -- THE NUMBER OF
STUDIES FIRST IN HUMANS AS EARLY
AS THE 2011 ONE, BUT THIS HAS
BEEN COMMERCIALIZED, THERE'S A
CAN BE USING THIS
THERAPEUTICALLY AND IT'S BEING
USED IN A NUMBER OF DIAGNOSTIC
APPLICATIONS.
SO IT'S A RATHER
MULTI-FUNCTIONAL MULTI-MODAL
PLATFORM THAT REALLY GOT ITS
START FROM A CTSA PILOT AWARD.
AND I THINK IF THAT APPLICATION
HAD COME TO AN NIH STUDY SECTION
EARLY ON BACK IN 2007, IT
PROBABLY WOULD HAVE BEEN NOT
DISCUSSED AT ALL.
LAST ONE I WANT TO FINISH WITH
IS AN INTERESTING ANALYSIS.
THIS IS A BIBLIOMETRIC ANALYSIS
THAT WAS DONE BY A NUMBER OF
CTSA AWARDEES.
AND THEY LOOKED AT ALL THE
CTSAs, I COULDN'T HAVE TOLD
YOU THIS NUMBER BEFORE THEY TOLD
ME WHAT IT WAS.
BUT FROM 2006 WHEN THE PROGRAM
BEGAN THROUGH 2021, THERE'S OVER
118,000 PUBLICATIONS THAT LIST
CTSA FUNDING WHICH IS IMPRESSIVE
IN AND OF ITSELF.
WHAT'S INTERESTING IS 13% OF
THOSE PUBLICATIONS HAVE BEEN
FOUND TO BE REFERENCED IN POLICY
DOCUMENTS WHICH IS A PRETTY
IMPRESSIVE NUMBER.
AND BY DOING AN ANALYSIS FOR THE
TYPE OF HEALTH-RELATED
PUBLICATIONS THAT THEY HAD
DESCRIBED, THAT'S ABOUT A 30%
INCREASE OVER NON-CTSA
PUBLICATIONS.
SO THE CTSA IS REALLY PRODUCING
STUDIES THAT ARE BEING READ NOT
JUST BY THE ACADEMIC COMMUNITY
AND INTERESTED INVESTIGATORS BUT
IS BEING REVIEWED AND EVALUATED
BY POLICYMAKERS AND PEOPLE IN
CHARGE OF ASSEMBLING CLINICAL
GUIDANCE AND THE LIKE.
THE OTHER INTERESTING ASPECT IS
THERE'S THIS LITTLE -- THIS
NIH-DERIVED APT, WHICH STANDS
FOR -- IT'S BIZARRE, NIH
PUBLICATION, IT'S APPROXIMATE
POTENTIAL TO TRANSLATE, IT'S NIH
SO A BIZARRE SORT OF NAME.
IT'S AN ML PREDICTION ALGORITHM
THAT ESTIMATES THE ABILITY OF A
PUBLICATION TO BE SUBSEQUENTLY
CITED IN A FUTURE CLINICAL STUDY
OF SOME TYPE.
AND WHAT THEY SHOWED WAS THAT
THE CTSA PUBLICATIONS THAT WERE
REFERENCED IN POLICY DOCUMENTS
HAD A STATISTICALLY HIGHER
LIKELIHOOD OF APTITUDE.
YOU LOOK AT SIMPLE SCIENTIFIC
STUDY AND WONDER IS ANYBODY
GOING TO DO SOMETHING USEFUL
WITH THIS LATER ON, PARTICULARLY
IN A CLINICAL AS OPPOSED TO
FOLLOW-ON BASIC SCIENCE STUDY.
THE APT IS AN ATTEMPT TO DO
THAT.
CTSA PUBLICATIONS VALIDATE THE
UTILITY OF THAT APT MEASURE.
I WILL CLOSE AND HAPPY TO TAKE
QUESTIONS.
>> YOU'RE TEN MINUTES OVER SO WE
HAVE -- I'D LIKE TO MAKE SURE WE
HAVE AN OPPORTUNITY TO HAVE
MAYBE A QUESTION OR SO.
IF THERE IS A QUESTION PLEASE GO
AHEAD.
IF NOT WE'LL MOVE ON.
GO AHEAD, PAUL.
>> I DON'T HAVE A QUESTION BUT
THAT WAS A PHENOMENAL
PRESENTATION.
THANK YOU.
>> THANK YOU.
>> TOUR DU FORCE.
>> THANK YOU.
>> HEAR, HEAR.
>> A CURIOSITY QUESTION.
WHERE YOU ENDED ON POLICY I'M
CURIOUS HOW MANY OF THE COVID
POLICIES, YOU TALK ABOUT RAPID
RESPONSE, WE HAVE ALL --
PROBABLY DON'T KNOW THE SUBSET
BUT INTERESTING TO SEE.
>> THIS ANALYSIS, I MEAN, I
THINK THIS PUBLICATION CAME OUT
IN 23, THINGS PUBLISHED THROUGH
21, SO THERE WOULD HAVE BEEN
VERY LITTLE OPPORTUNITY I THINK
FOR COVID.
I HAVE TO SAY IT IS NOT TRIVIAL.
WE'VE TRIED TO DO THIS AND
TRACKING WHAT APPEARS IN POLICY
DOCUMENTS IS NOT AN EASY TASK.
AND IT'S SOMETHING WE'RE TRYING
TO PUT MORE EFFORT INTO.
IT'S NOT STRAIGHTFORWARD, NOT
SIMPLE.
>> YOU CAN DO IT EASIER WITH
(INDISCERNIBLE) DOCKET.
>> THAT'S ONE -- YES.
BUT THERE'S AN -- I MEAN, YOU
KNOW, WE'VE SEEN EXAMPLES OF
WHERE, YOU KNOW, FINLAND HAS
CITED CTSA PUBLISHING
INDICATIONS IN THEIR POLICY
DOCUMENTS, SO W.H.O.
EXTENSIVELY.
IT'S NOT JUST U.S.
IT'S NOT JUST PROFESSIONAL
GUIDANCE OR CDC OR SOMETHING
LIKE THAT.
IT'S FAR MORE.
IT'S A BIG LIFT.
>> THERE HAS BEEN SOME
REFERENCES TO THINGS THAT HAVE
BEEN HAPPENING THROUGH COVID
LIKE LESSONS LEARNED FROM THE
PANDEMIC, ALSO CTSA PROGRAM
RELATED TO N3C FOR EXAMPLE,
THERE HAVE BEEN THOSE THINGS IN
TERMS OF DEVELOPING DOCUMENTS
FOR PREPAREDNESS FOR FUTURE
PANDEMICS, FROM THE WHITE HOUSE.
SO THERE ARE SOME ASPECTS OF
THOSE.
YEAH, PROBABLY MORE THAN WE
REALIZE.
ALL RIGHT.
THANK YOU.
AND WE HAVE ONE MORE PROGRAM
UPDATE FROM DR. CHRISTINE COLVIS
FROM OFFICE OF DRUG DEVELOPMENT
PARTNERSHIP PROGRAM.
AND SHE WILL GO AHEAD AND TAKE
OVER.
>> OKAY.
THANK YOU.
YEP, I'M CHRISTINE COLVIS, I'LL
GIVE AN UPDATE ON SOME OF THE
PROGRAMS THAT WE HAVE GOING
RIGHT NOW IN OUR OFFICE.
I'LL CEDE TIME TO DR. BECK FOR A
MORE DETAILED UPDATE ON THE
TRANSLATOR PROGRAM.
THIS IS SHOWING YOU AN OVERVIEW
OF THE DIFFERENT THINGS WE HAVE
GOING ON.
WE COVER PRE-CLINICAL
DEVELOPMENT AND INTO EARLY STAGE
CLINICAL TRIALS BUT WE ALSO HAVE
THINGS THAT SPAN THE ENTIRE
PIPELINE SO WE PRIMARILY ARE
FOCUSED ON EITHER DEVELOPING
MORE TREATMENTS OR MORE QUICKLY,
AND DON'T TOUCH SO MUCH ON ALL
PEOPLE EXCEPT CARLY DOES AND
SHE'LL TELL BUT A CONCEPT LATER
TODAY WHERE SHE TRIES TO ADDRESS
THE ALL-PEOPLE ASPECT OF OUR
MOTTO.
EXPANDING THE TARGET LANDSCAPE,
SO, THIS WAS REALLY INITIATED BY
AN NIH COMMON FUND PROGRAM
CALLED THE ILLUMINATING THE
DRUGGABLE GENOME PROGRAM.
AND WITH NIH COMMON FUND
PROGRAMS THEY INVOLVE LARGE
CENTERS, ADMINISTRATIVE CORE,
ALL THOSE THINGS.
THE PROGRAM MANAGED BY PROGRAM
STAFF FROM ACROSS THE NIH,
USUALLY WITH ONE OR TWO PROGRAM
PEOPLE WHO OVERSEE THE ENTIRE
THING FOR THE NIH.
KARLIE DOES THAT FOR
ILLUMINATING THE DRUGGABLE
GENOME PROJECT, OVERSEES THE
WHOLE THING.
I WON'T TELL YOU ABOUT THE LARGE
CENTERS AND EVERYTHING.
WHAT I WANT TO TELL BUT IS A
SMALL GRANT PROGRAM SHE STARTED
IN THE COMMON FUND ELIMINATING
THE DRUGGABLE GENOME PROGRAM.
FIRST LET ME REMIND YOU OF WHAT
THE IDG PROGRAM IS ABOUT.
THE GOAL TO UNDERSTAND THE
PROPERTIES AND FUNCTIONS OF
UNDERSTUDIED PROTEINS WITHIN
DRUGGABLE PROTEIN FAMILIES.
FOR BUDGETARY REASONS WE LIMITED
DRUGGABLE PROTEIN FAMILIES TO
ION CHANNELS, GPCRs, KINASES.
THERE ARE MANY MORE.
BUT AGAIN THAT WAS LIMITED.
WHEN WE TALK ABOUT UNDERSTUDIED
PROTEINS WE MEAN PROTEINS THAT
EITHER HAVE NO PUBLICATIONS OR
VERY FEW PUBLICATIONS AND ALSO
LACK R01 FUNDING.
THESE ARE LOW-HANGING FRUIT,
POTENTIALLY, FOR THERAPEUTIC
DEVELOPMENT.
YET NOBODY'S REALLY LOOKING.
THE IDEA HERE IS TO CEDE
RESEARCH ON THESE LOW-HANGING
FRUIT, UNDERSTUDIED PROTEINS,
THAT WILL EVENTUALLY LEAD ON TO
MORE INVESTIGATION IN THE
FUTURE.
SO WHAT KARLIE DID WAS DECIDED
TO START A SMALL GRANT PROGRAM,
R03 PROGRAM, JUST $100,000 A
YEAR DIRECT COST FOR JUST ONE
YEAR.
THE IDEA JUST TO SEED AND
PROVIDE ENOUGH INFORMATION FOR
SOMEBODY TO GATHER PRELIMINARY
DATA, DECIDE WHETHER IT'S WORTH
PURSUING AND SUBMIT AN
APPLICATION FOR A LARGER AWARD.
THIS HAS BEEN VERY SUCCESSFUL.
THE FIRST AWARDS WERE MADE IN
2019.
SHE'S MADE OVER -- ALMOST 100
AWARDS THUS FAR, AND ABOUT A
THIRD OF THESE ACTUALLY END UP
BEING AWARDED TO EARLY-STAGE
INVESTIGATORS AND NEW
INVESTIGATORS, THIS IS A
BY-PRODUCT OF HAVING THIS
SMALLER AWARD PROGRAM AND SO
THIS IS REALLY ATTRACTIVE TO
EMERGING SCIENTISTS IN THE
COMMUNITY AND HELPING HOPEFULLY
TO ALSO BUILD THAT WORKFORCE AND
GIVE THEM A NICHE TO SORT OF
BUILD ON AND HAVE AS THEIR OWN
SPACE.
THE REAL PROOF IN THE PUDDING IS
IN WHETHER OR NOT THIS SEED GETS
NURTURED AND GROWS AND DOES GO
ON TO LARGER AWARDS, R01s,
R21s, THAT TYPE OF THING.
WE DO SEE THAT.
IT TAKES OF COURSE A LITTLE BIT
OF TIME BUT IF WE LOOK AT, YOU
KNOW, THREE TO FOUR YEARS AFTER
THE INITIAL R03 AWARD IS MADE,
WE SEE 40 TO 50% RECEIVE AN R01
AND R21, COOPERATIVE AGREEMENT
AWARD OR SOMETIMES AN AWARD
THAT'S OUTSIDE OF THE NIH EVEN.
BUT IN THIS RESPECT WE FEEL LIKE
WE ARE STARTING TO EXPAND THE
CAR GET LANDSCAPE, WE'RE GIVING
PEOPLE NEW TARGETS TO GO AFTER
FOR DEVELOPING DRUGS.
AND WITH VERY, VERY MODEST
SUPPORT.
SO, SEEING THAT SUPPORT, THAT
WAS NIH COMMON FUND MONEY, NIH
O.D. MONEY.
SEEING THAT SUPPORT WE THOUGHT
MAYBE WE SHOULD DO SOMETHING FOR
RARE DISEASES AND ACTUALLY DO
SOMETHING, YOU KNOW, USING NCATS
MONEY.
SO WE ESTABLISHED A SIMILAR
PROGRAM WHERE THE APPLICATIONS
HAVE TO BE FOCUSED ON AN
UNDERSTUDIED PROTEIN, WE PROVIDE
A LIST OF THE QUALIFYING
PROTEINS.
THESE ARE AGAIN DRUGGABLE
PROTEINS THAT ARE ASSOCIATED
WITH A RARE DISEASE.
SO IT'S UP TO THE INVESTIGATOR
TO MAKE THAT ASSOCIATION.
AGAIN, STILL ONLY PROVIDING
$100,000 FOR JUST ONE YEAR,
KARLIE MANAGED TO GARNER SUPPORT
FROM ALSO OTHER INSTITUTES
ACROSS THE NIH AND IN THIS PAST
YEAR WHICH IS OUR FIRST YEAR OF
ACTUALLY HAVING THESE AWARDS, 21
AWARDS WERE MADE, THIS FISCAL
YEAR.
WE HAVE UPCOMING RECEIPT DATE IN
A COUPLE WEEKS, ONE IN JANUARY
AND JULY.
WE ENCOURAGE PEOPLE TO TAKE A
LOOK IF THERE'S A RARE DISEASE
THEY ARE INTERESTED IN.
I WANT TO TELL YOU ABOUT A
PROGRAM ADDRESSING THE MORE
QUICKLY PART OF OUR MOTTO, THE
ASSETS PROGRAM BEING LED BY DR.
CONGRESS.
DR. KANG.
THIS IS A SMALL AWARD PROGRAM,
R21s TO SUPPORT EARLY STAGE
TECHNOLOGY DEVELOPMENT TO
IMPROVE EFFICIENCY ALONG THE
TRANSLATIONAL PIPELINE, WE WANT
TO DOUGH THIS -- DO THIS IN A
DISEAS AGNOSTIC WAY BECAUSE
WE'RE NCATS.
WE WANT A FUTURE PLAN FROM
INVESTIGATORS, WE WANT THEM
THINKING ABOUT HOW IS THIS
TECHNOLOGY GOING TO BE USED IN
THE REAL WORLD, HOW IS IT GOING
TO GET TO THE REAL WORLD AND GET
TO THAT POINT.
IT'S NOT QUITE A
COMMERCIALIZATION PLAN LIKE SBIR
BECAUSE THESE ARE VERY EARLY
STAGES.
WE DON'T THINK AFTER TWO YEARS
OF AN R21 SUPPORT THEY WILL HAVE
SOMETHING COMMERCIALIZABLE BUT
WE ASKED FOR THAT FUTURE PLAN.
THE FIRST RECEIPT DATE WAS JUST
THIS PAST JULY.
IT'S REALLY, REALLY EARLY DAYS.
THERE'S NOT A LOT I CAN TELL
YOU, BUT I CAN SHOW YOU SORT OF
WHAT THE CHARACTERISTICS ARE OF
THE APPLICATIONS WE RECEIVED.
WE SEE A REALLY NICE
DISTRIBUTION OF TECHNOLOGIES,
AND WE ALSO SEE A NICE
DISTRIBUTION ACROSS THE STAGES
ALONG THE TRANSLATIONAL PIPELINE
THAT WE'RE SEEING.
I'LL TELL YOU, WE HAD - TO SAY A
ROBUST RESPONSE IS AN
UNDERSTATEMENT.
WE HAD A VERY, VERY STRONG
RESPONSE TO THIS, THIS FUNDING
OPPORTUNITY.
THAT'S A REALLY GOOD THING
BECAUSE IT MEANS WE'VE ACTUALLY
HIT ON SOMETHING WHERE THERE WAS
A REAL NEED, BUT WE ARE
CONCERNED THAT GIVEN NCATS'
BUDGET RIGHT NOW WE WOULDN'T BE
ABLE TO SUPPORT AS MANY AS WE
WOULD LIKE TO SO WE MAY BE
NARROWING THE SCOPE OF THIS
ANNOUNCEMENT GOING FORWARD IN
FAIRNESS TOP APPLICANTS, WE
DON'T WANT IT SO COMPETITIVE THE
CHANCE OF GETTING AN AWARD ARE
TOO SLIM.
THAT SAID, WE DID HAVE TWO MORE
RECEIPT DATES.
KEEP AN EYE ON THIS.
WE MIGHT BE TWEAKING THINGS.
BUT AS OF RIGHT NOW WE HAVE
RECEIPT DATE ONCE A YEAR, JUNE
19, OF BOTH 2024 AND 2025, AGAIN
BUDGETS ARE STANDARD.
IN TERMS OF R01 -- SORRY,
R21-TYPE AWARDS.
NOW, ANOTHER PROGRAM THAT I WANT
TO TELL BUT VERY QUICKLY IS THE
LITCOIN PROGRAM.
THE MAIN GOALS OF THIS PROGRAM
WITH TO INCENTIVIZE DATA AND
KNOWLEDGE SHAKER, THAT'S THE
LITCOIN PROPER.
THE SECOND BULLET HERE IS TO
BUILD MACHINE READABLE
A.I.-READY KNOWLEDGE FROM FREE
TEXT AT THE TIME OF THE
MANUSCRIPT SUBMISSION.
AND I WANT TO FOCUS ON THAT
SECOND BULLET.
SO THAT SECOND BULLET IS MUCH
BROADER THAN JUST A LITCOIN.
A LITCOIN AS YOU MAY RECALL IS
SOMETHING VERY -- A SPECIFIC
TYPE OF PUBLICATION LIKE A
MICROPUBLICATION, BUT THE SECOND
BULLET IS SOMETHING THAT WE SEE
AS SOMETHING THAT CAN BE
APPLIED TO ANY PUBLICATION GOING
FORWARD.
THE AUTHOR WOULD SUBMIT TO A
LITCOIN PORTAL YET TO BE BUILT,
WOULD SUBMIT THEIR ABSTRACT FOR
THE PAPER, TO THE PORTAL, THE
PORTAL RUNS NATURAL LANGUAGE
PROCESSING AND GENERATES A
MACHINE READABLE KNOWLEDGE GRAPH
OR NETWORK THAT REPRESENTS THE
TEXT THAT WAS IN THE ABSTRACT.
THE REASON YOU WANT TO DO THAT
IS BECAUSE THAT GRAPHICAL
REPRESENTATION, THAT NETWORK
REPRESENTATION, IS MUCH EASIER
FOR THE COMPUTER TO ACTUALLY
UNDERSTAND AND EXTRACT KNOWLEDGE
FROM.
WHEN THEY SUBMIT THEIR
MANUSCRIPT TO THE PUBLISHER THEY
WOULD ALSO SUBMIT THIS GRAPH.
THE PUBLISHER THEN WOULD TAKE
THAT AND PASS IT ON TO THE PEER
REVIEWERS, ALONG WITH THE
MANUSCRIPT.
THEY ARE ALSO GOING TO LOOK AT
THIS GRAPH.
I SHOULD SAY THE AUTHOR WHEN THE
GRAPH IS GENERATED, THE AUTHOR
HAS AN OPPORTUNITY TO MAKE
CORRECTIONS, TO SAY, NO, THE
ARROW HAS TO BE TURNED AROUND.
THE REVIEWERS THEN HAVE AN
OPPORTUNITY TO CONFIRM THAT,
YES, THIS GRAPH, CORRECTED
GRAPH, DOES REPRESENT WHAT IS
BEING SAID IN THE TEXT OF THE
PAPER.
SO WHEN THE MANUSCRIPT IS
ACCEPTED FOR PUBLICATION, THAT
GRAPH GETS SENT BACK TO THE
LITCOIN PORTAL.
AND IT GETS INCORPORATED INTO
THIS MACHINE READABLE
FOUNDATIONAL GRAPH WHICH
REPRESENTS THE CORPUS OF
KNOWLEDGE THAT WE ALREADY HAVE
THAT'S IN THIS MACHINE READABLE
FORM.
AND SO AS YOU CAN SEE OVER TIME
YOU JUST BEGIN TO BUILD THIS.
SO THIS IS SOMETHING THAT WE
WANT TO SEE HAPPEN WITH ALL
PUBLICATIONS.
WE WERE ADVISED WE SHOULD PILOT
THIS FIRST.
SO WE'RE PILOTING IT.
I'LL SAY MORE IN A SECOND.
AS YOU CAN SEE THIS PROCESS IS
VERY NEW, A NEW CONCEPT.
WE PUT OUT A PRIZE COMPETITION,
A CHALLENGE, TO HEAR WHAT
PEOPLE'S IDEAS WERE IN TERMS OF
HOW YOU WOULD DESIGN THE SYSTEM
THAT WOULD ACTUALLY ALLOW THIS
TO HAPPEN, AND TO GIVE US THE
TECHNICAL REQUIREMENTS THAT
WOULD BE NEEDED IN ORDER FOR
THIS TO ACTUALLY WORK.
WE HAVE A VISION BUT WE NEED TO
KNOW WHAT ARE GOING TO BE OUR
TECHNICAL REQUIREMENTS IF WE
WANT TO MAKE THIS HAPPEN.
WE ENDED UP AWARDING TWO FIRST
PRIZES AND SECOND PRIZE, ALL
THREE GREAT BUT WE WENT WITH TWO
FIRST PRIZEs INSTEAD OF FIRST,
SECOND, THIRD, BECAUSE THE FIRST
TEAM YOU'RE SEEING WAS VERY
FOCUSED ON THE NEEDS OF THE
PUBLISHER.
THE SECOND TEAM WAS VERY FOCUSED
ON THE NEEDS OF THE AUTHORS.
AND ONE OF THE ADVANTAGES OF
USING THE PRIZE MECHANISM IS NOW
WE CAN ACTUALLY TAKE THE BEST OF
BOTH WORLDS AND DESIGN A SYSTEM
THAT'S REALLY HOPEFULLY GOING TO
BE, YOU KNOW, WORK FOR
EVERYBODY.
AND THEN THE OTHER PIECE THAT'S
IMPORTANT FOR US TO DO EARLY ON
IS ESTABLISH THAT FOUNDATIONAL
KNOWLEDGE GRAPH, AND WE'RE DOING
THAT WITH FINANCIAL SUPPORT FROM
THE HEAL PROGRAM, HELPING END
ADDICTION LONG TERM.
WE'LL FOCUS OUR FOUNDATIONAL
GRAPH IN THAT SPACE, SO IN PAIN
AND ADDICTION.
AND WE AWARDED CONTRACT JUST
LAST WEEK TO THE RTI
INTERNATIONAL TO HELP US TO
BUILD THAT.
THE LAST BIT OF UPDATE FOR
LITCOIN IS THAT WITH SUPPORT
FROM "HEAL," AND FROM THE
OFFICES OF DATA SCIENCE STRATEGY
WE'RE GETTING SUPPORT FOR DR.
AARON BERNSTEIN TO WORK WITH US
AS AN ODSS DATA SCHOLAR, AARON
IS A NEWLY MINTED PhD and just
started on Monday but he's
really hitting the ground
running.
WE'RE VERY EXCITED TO HAVE HIM.
THE ONE OTHER THING I WANTED TO
TELL YOU ABOUT BEFORE I HAND
THIS OFF TO TYLER IS A
COLLABORATION THAT KARLIE
ESTABLISHED WITH CHRISTINE
COTILLO IN THE NCATS O.D. AND
HAS GONE ON TO THE OFFICE OF
DATA SCIENCE STRATEGY, NIH O.D.,
WE'RE HAPPY FOR HER, SAD FOR US.
BUT BEFORE SHE LEFT SHE AND
KARLIE ISSUED A CHALLENGE, PRIZE
COMPETITION, FOCUSED ON
MINIMIZING BIAS, MAXIMIZING
LONG-TERM ACCURACY OF PREDICTIVE
ALGORITHMS AND HEALTH CARE, AND
I WANTED TO ACKNOWLEDGE THE
FIRST PLACE TEAM, TEAM INTERFAIR
THAT WON, WITH THE MOST
COMPREHENSIVE SOLUTION FOR THIS
AND VERY EXCITED FOR THEM.
OKAY.
WITH THAT, I'M GOING TO INVITE
TYLER TO COME UP TO GIVE YOU AN
UPDATE ON TRANSLATOR AND I'M
GOING TO INVITE OUR PANELISTS TO
COME AND SIT AT THE TABLE.
AFTER TYLER'S UPDATE, HE'S GOING
TO GO STRAIGHT INTO THE CONCEPT
CLEARANCE THAT HE HAS FOR THAT.
MAYBE WE'LL WANT TO PAUSE FOR
QUESTIONS BEFORE HE GOES TO THE
CONCEPT CLEARANCE.
>> WE SHOULD HAVE AN OPPORTUNITY
FOR QUESTIONS.
>> OKAY.
WE'LL PAUSE FOR QUESTIONS.
>> IF YOU HAVE A POSITIVE
RESPONSE DON'T SHUT THAT
RESPONSE DOWN.
WHAT YOU DO IS EVEN DON'T TELL
YOU, YOU PANEL, THEY CAN ONLY
FUND 5% BECAUSE THEY WILL BE
EXTRA CRUEL TO THE GRANTS COMING
IN BECAUSE THEY HAVE TO BE
REALLY (INDISCERNIBLE).
DON'T DO THAT.
TAKE THOSE SCORES TO THE
DIRECTOR OF THE INSTITUTE AND
SHE WILL HAVE THE FUN JOB TO
DECIDE WHAT HAS TO HAPPEN.
I'M SERIOUS.
OTHER INSTITUTES ARE DOING THAT.
IF YOU HAVE A BRANCH IN YOUR
INSTITUTE WHO IS ABLE TO
MOBILIZE RESEARCH COMMUNITY, I
TELL MY MENTEES NOT TO, BECAUSE
THEY ARE AS MUCH WORK AS AN R01
AND YOU GET TWO YEARS OF
FUNDING, SO IT'S A FACT YOU'VE
GOT SUCH AN OVERWHELMING
RESPONSE REALLY TELLS YOU
(INDISCERNIBLE) SO PLEASE DON'T
SHUT IT DOWN.
FIND CREATIVE WAYS LIKE
REALLOCATING FUNDS, LOOKING AT
FOUNDATIONAL FUNDS TO SEE WHERE
CAN YOU LEVERAGE.
>> YEAH, THANK YOU.
THANK YOU.
>> ALL RIGHT.
I'M TYLER BECK.
I'M GOING TO TALK ABOUT THE
PROGRAM THAT I'VE BEEN WORKING
ON SINCE I STARTED NCATS FIVE
YEARS AGO NOW.
AND SO THIS IS THE BIOMEDICAL
DATA TRANSLATOR.
I KNOW SEVERAL OF YOU HAVE SEEN
PRESENTATIONS ON THIS BEFORE BUT
I WANT TO GIVE A BACKGROUND ON
IT.
SO, TRANSLATOR IS DESIGN TO HELP
SURFACE KNOWLEDGE FROM A VAST
ARRAY OF BIOMEDICAL DATA.
AUGMENTING THROUGH THE USE OF
KNOWLEDGE GRAPHS, HUGE GROUPS OF
DATA POINTS CONNECTED THROUGH
RELATIONSHIPS.
THESE ARE ENCODED INTO THIS
LARGE CLOUD, INFORMATION LIKE
THIS TRAVERSES DATA AND PRESENTS
RELATIVE CONNECTIONS BETWEEN THE
PIECES OF INFORMATION.
THESE CONNECTIONS REPRESENT
RELATIONSHIPS FOUND IN MANY
SOURCES INCLUDING THOSE
SUPPORTED BY NIH LIKE PubMed
AND GUARD AND PubChem, ALONG
WITH PUBLIC DATABASES NOT
SUPPORTED DIRECTLY BY NIH SUCH
AS SNOMED AND MANY OTHERS.
IT'S WORTH NOTING GETTING THESE
DISPARATE DATA SOURCES TO
INTERACT WITH EACH OTHER IN A
COHERENT WAY WAS A HUGE TASK
TRIED BY MANY OTHER GROUPS
BEFORE WITH LITTLE SUCCESS.
HAVING A SINGLE LANGUAGE TO
DESCRIBE CONCEPTS ACROSS ALL OF
BIOMEDICAL SCIENCE IS A HUGE
UNDERTAKING.
WE'VE CREATED SEVERAL STANDARDS
AS PART OF THIS CONSORTIUM OVER
THE LAST FEW YEARS.
SOME OF WHICH ARE USED IN OTHER
LARGE DATA SCIENCE PROGRAMS AS
WELL.
SO AS YOU'RE PROBABLY AWARE
TRANSLATOR IS A SYSTEM SOFTWARE
TOOLS BUILT BY 14 TEAMS.
WE HAVE SEVERAL TYPES OF TEAMS
WITH DIFFERENT FUNCTIONALITY.
FIRST OFF WE HAVE KNOWLEDGE
PROVIDER TEAM, TASKED WITH
SEARCHING FOR AND COLLECTING
DATA FROM DATABASES AROUND THE
WEB.
DATABASES CAN ANSWER THE LOWEST
COMPLEXITY QUESTIONS THAT USERS
MIGHT HAVE FOR A SYSTEM ON THEIR
OWN SUCH AS WHAT CELL TYPE A
GENE IS HIGHLY EXPRESSED IN.
THEY BUILD KNOWLEDGE GRAPHS AND
SERVE AS EXPERT SOURCE FOR THAT
TYPE OF KNOWLEDGE.
WE HAVE GENOMICS DATA PROVIDER,
CLINICAL DATA PROVIDER, CHEMICAL
DATA PROVIDER AND SEVERAL
OTHERS.
WE HAVE ARAs WHICH ACCESS
GRAPHS, REQUESTING SPECIFIC
PARTS OF THE UNDERLYING
KNOWLEDGE GRAPHS BASED ON WHAT
THE USER ASKED.
THEY COMBINE THESE RESULTS INTO
A NEW GRAPH WHICH INCLUDES
KNOWLEDGE FROM MANY DOMAINS
CONNECTED TOGETHER.
ARA WILL FIND A PATH THAT MAY
SATISFY THE CRITERIA OF THE
QUESTION ASKED.
SO THESE RESULTS ARE RANKED
BASED ON THINGS LINING
CONFIDENCE SCORES AND METRICS
AND EACH HAS A DIFFERENT WAY OF
RANKING THEIR RESULTS.
EACH OF THESE ARE THEN SENT TO
THE AUTONOMOUS RELAY SYSTEM
WHICH THE INTRAMURAL TEAM BUILT
FOR US.
ALL THESE POTENTIAL RESULTS ARE
SORTED AND COMBINED AS NECESSARY
INTO A SET OF POTENTIAL ANSWERS
FOR THE USER.
OVER THE COURSE, WE'VE MADE
STRIDES TOWARDS MAKING THE
SYSTEM A REALITY AND NOW AN
ALPHA VERSION OF USER INTERFACE,
INTERACTING WITH THE ARAS TO
DISPLAY RULEDS TO EXPLORE
EVIDENCE AND SOURCE OF RESULTS,
BUILT THROUGH A CONTRACT WITH A
COMPANY CALLED DMI WHO WORKED
WITH TRANSLATOR TEAMS AND USER
GROUPS TO DESIGN FRONT END FOR
TRANSLATOR.
WE'LL LOOK AT INTERFACE IN A FEW
MOMENTS.
THEY HAVE TO TALK TO EACH OTHER.
THIS IS THE DIAGRAM FROM THE
JUNE HACKATHON MEETING.
YOU CAN SEE SOMETHING THAT'S
COMPLICATED WHICH DOESN'T
ACCOUNT FOR ACCESSING AND
UPDATING KNOWLEDGE GRAPHS
KNOWLEDGE PROVIDERS THEMSELVES
STORE.
THIS IS JUST THE INFORMATION
THAT NEEDS TO FLOW BETWEEN OUR
TOOLS WHEN A QUERY IS MADE.
THIS GIVES A TASTE OF HOW MUCH
WORK HAS GONE INTO THE
ARCHITECTURE AND DEVELOPMENT OF
THE SYSTEM UP TO THIS POINT.
A LITTLE HISTORY ON THE PROJECT,
I KNOW SOME OF YOU ARE FAMILIAR
WITH IT.
FEASIBILITY WAS ACHIEVED IN
2019.
WE BEGAN THE CURRENT DEVELOPMENT
PHASE OF THE PROGRAM.
WE BEGAN WITH COMPETITIVE
PROTOTYPING PHASE, IN JANUARY OF
2020, PART OF THE FINAL
APPLICATION PROCESS FOR THIS
PHASE.
AND ONCE WE CHOSE OUR WINNERS
BEGAN FULL DEVELOPMENT OF THE
SYSTEM IN MAY OF THAT YEAR.
THAT MEANS RIGHT NOW WE'RE JUST
OVER 3 YEARS INTO DEVELOPMENT OF
THE SYSTEM.
IN ADDITION TO DEVELOPMENT
RESEARCH TEAMS WE HAVE THE USER
INTERFACE DEVELOPMENT TEAM, AND
THEY ARE TWO YEARS INTO A
THREE-YEAR FUNDING PERIOD.
SORRY.
GO BACK.
THERE WE GO.
WE WERE ABLE TO BUILD UPON THE
STANDARDS AND SOME GRAPHS
CREATED DURING FEASIBILITY BUT
FOR THE MOST PART THE
DEVELOPMENT PHASE OF TRANSLATOR
REPRESENTED BRAND NEW START FOR
THE CONSORTIUM.
WITH SEVERAL BRAND NEW TEAMS AND
MANY OF THE PREVIOUS TEAMS
SELF-REORGANIZING INTO
COLLABORATIONS DURING THE
PROCESS.
WE'VE BEEN HOLDING HACKATHON
TYPE MEETINGS, UP UNTIL RECENTLY
HAD BEEN VIRTUAL BECAUSE OF
COVID CONCERNS BUT IN JUNE WE
HELD OUR FIRST IN-PERSON
HACKATHON IN OVER FOUR YEARS.
AT THE BEGINNING OF THIS MONTH
WE RELEASED OUR FIRST PUBLIC
ALPHA VERSION OF THE TRANSLATOR
SYSTEM AND CURRENT VERSION
INCLUDES FUNCTIONALITY FOR USERS
TO ASK A SET OF SPECIFIC
QUESTIONS OF THE SYSTEM AND GET
BACK POTENTIAL ANSWERS TO THESE
QUESTIONS ALONG WITH THE
EVIDENCE AND PROVENANCE
ASSOCIATED WITH EACH ONE.
WE'VE BEEN HOLDING HACKATHONS,
RELAY MEETINGS, TWO TO THREE
TIMES A YEAR, TRUE WORKING
MEETINGS.
EVERY SESSION HAS TO HAVE
DELIVERABLES TO FIND SO WE CAN
KEEP TRACK OF ALL THAT WORK.
MEETINGS WERE VIRTUAL FOR
SEVERAL YEARS BECAUSE OF COVID
BUT NOW WE STARTED HAVING
IN-PERSON COMPONENT TO MEETINGS
WHEN POSSIBLE.
WE'VE PERFECTED VIRTUAL MEETINGS
AS NECESSITY IN TRANSLATOR
PROGRAM, IN-PERSON MEETING STILL
HAS ADVANTAGES.
ONE OF WHICH IS OUR TEAMS ARE
HIGHLY MOTIVATED AND YOU CAN
OFTEN FIND AT LEAST A SMALL
GROUP WORKING LATE INTO THE
NIGHT IN THE HOTEL LOBBY ON SOME
IDEA THEY CAME UP WITH, AND
THAT'S HARD IF NOT IMPOSSIBLE TO
REPLICATE IN AN ALL-VIRTUAL
SETTING.
ON THE TOP RIGHT SHOWS A GROUP
WORKING ON SOFTWARE AT MIDNIGHT.
BELOW THAT IS PRESENTATION ABOUT
IT THE NEXT DAY TO THE FULL
GROUP.
MOST RECENT MEETING IN POTOMAC
LAST WEEK TESTED THE IDEA
ALLOWING FOR AD HOC WORKING
SESSION WAS BREAKOUT ROOMS
ESSENTIALLY AVAILABLE WHENEVER
THEY WEREN'T BEING USED FOR
SCHEDULED SESSION, AND THIS
ALLOWED SMALL GROUPS, WORKING
SESSIONS IN A MORE FORMALIZED
WAY.
SO LET'S TALK ABOUT WHO WE'RE
BUILDING THE SYSTEM FOR.
WE EXPECT MAIN USER BASE WILL BE
TRANSLATIONAL RESEARCHERS,
PARTICULARLY RARE DISEASE
RESEARCHERS.
IT MAY BE THAT THEY ARE IN THE
LAB AND OBSERVED SOMETHING
INTERESTED, LOOKING FOR
POTENTIAL EXPLANATIONS OR
SUPPORT BEFORE THEY PURSUE IT
FURTHER.
OR MAY BE THESE ARE EARLY CAREER
RESEARCHERS WITH EXCITING NEW
IDEAS, LOOKING FOR MECHANISMS
THAT MIGHT SUPPORT THEIR
HYPOTHESES.
WE HAVE A BUILT-IN GROUP OF
USERS WHO HAVE GIVEN US FEEDBACK
DURING THIS INITIAL STAGE,
ANALYSTS WORKING AT THE
PRECISION MEDICINE INSTITUTE AT
THE UNIVERSITY OF ALABAMA
BIRMINGHAM.
THESE WORKED DIRECTLY WITH
CLINICAL RESEARCHERS LOOKING FOR
NEW POTENTIAL TREATMENTS FOR
VERY RARE OR REFRACTORY
CONDITIONS.
I'LL TALK MORE ABOUT HOW THAT
PROCESS WORKS IN A BIT.
WE HAVE ONE MORE VERY DIFFERENT
GROUP OF USERS FOR TRANSLATOR,
DATA SCIENTISTS COLLABORATOR WHO
WISH TO CONTRIBUTE DATA AND USE
KNOWLEDGE GRAPHS THROUGH APIs
AND MECHANISMS OF INTERACTING
WITH THE GRAPHS.
I MENTIONED ON THE TIMELINE
SLIDE WE JUST HAD OUR ALPHA
RELEASE.
I WANTED TO MAKE IT CLEAR WHAT
WE MEAN BY ALPHA RELEASE.
THIS IS A TERM USED FOR EARLY
INVESTIGATORS OF SOFTWARE,
UNSTABLE, USEFUL TO SHOW WHAT
THE SOFTWARE WILL DO.
THAT'S THE POINT WE'RE AT WITH
TRANSLATOR.
I WANT YOU TO KEEP THAT IN MIND
AS WE MOVE FORWARD.
OUR SYSTEM CAN SUPPORT ALMOST
ANY QUERY THROUGH THE API.
WE MADE THE DECISION TO START
WITH TEMPLATED QUERIES TO MAKE
SURE THE RESULTS ARE TAILORED TO
REPRESENT INFORMATION THAT USERS
REALLY WANT TO SEE.
AT THIS TIME A USER CAN ASK
THREE QUERIES.
WHAT DRUGS OR CHEMICALS MAY
TREAT A DISEASE OF INTEREST?
WHAT GENES MAY BE UP OR
DOWNREGULATED BY DRUG OR
CHEMICAL OF INTEREST?
AND WHAT DRUGS OR CHEMICALS MAY
UPOR DOWNREGULATE THEIR GENE OF
INTEREST?
I WANT TO MAKE THE NOTE HERE
THAT THE TERM DRUG IS USED
LOOSELY AND ANY CHEMICAL WHICH
MAY OR MAY NOT HAVE ANY KIND OF
FDA APPROVAL STATUS CAN BE
RETURNED IN THESE RESULTS ARE
OFTEN THE RESULTS THAT THE USERS
ARE REALLY LOOKING FOR.
WE USE A COMPLEX SYSTEM AND HAVE
A COMPLEX SCORING SCHEME THAT
HAD TO BE IMPLEMENTED, WE'RE
WORKING ON IMPROVEMENTS TO THE
SCORING SCHEME NOW.
AT THE MOMENT UTILIZES
COMBINATION OF WEIGHTED SCORES
COMING FROM EACH OF THE
REASONING AGENTS THAT RETURNED A
GIVEN RESULT.
WE'RE IN THE PROCESS OF
IMPLEMENTING ADDITIONAL SCORING
METRICS SUCH AS ASSESSMENT OF
NOVELTY OF A GIVEN ANSWER AND
INCLUDING DIRECT CLINICAL
EVIDENCE WHERE IT EXISTS.
SO THIS IS WHAT CURRENT USER
INTERFACE LOOKS LIKE, IMPORTANT
TO NOTE AT THE TOP OF THE PAGE
WE ALWAYS SHOW A BANNER THAT
IT'S FOR RESEARCH ONLY, NOT AS
DECISION SUPPORT IN THE COURSE
OF TREATMENT OF PATIENTS.
I'LL WALK YOU THROUGH A QUERY TO
SEE THE PROCESS FOR YOURSELVES.
I'LL USE GENES TO CHEMICALS
QUERY ON THE RIGHT SIDE GIVING
INFORMATION ON EFFECTS ON GENE
REGULATION.
WE EXPECT THIS WILL OFTEN BE
USED BY RESEARCHES LIKE THOSE IN
OUR OWN DPI, PRE-CLINICAL
DEVELOPMENT, USEFUL TO RESEARCH
RESEARCH TEAMS TO HELP NARROW
DOWN CHEMICALS TO SCREEN DURING
EARLY DEVELOPMENT.
WE NEED TO SPECIFY WE WANT
CHEMICALS CAN REGULATE A GENE.
WE'LL USE UPREGULATORS IN THIS
CASE.
A SEARCH BAR LETS THE USER KNOW
IT NEEDS AN ENTRY, WE'LL USE
FRAG 1.
WHEN YOU START TYPING THE NAME
THE SEARCH BAR WILL SHOW
OPTIONS.
CLICK ON ONE TO CONFIRM WHICH
GENE THEY ARE INTERESTED IN.
WHILE YOU'RE WAITING FOR
RESULTS, SYSTEM GIVES
INFORMATION ABOUT THE ENTITY YOU
SEARCHED FOR, THE IN THIS CASE
FRAS1 GENE.
WE'LL LOOK AT PATHS IN A MOMENT.
EVIDENCE THAT SUPPORTS IT
INCLUDING PUBLICATIONS AND
SCORE.
WE'LL TAKE A LOOK AT ONE
SPECIFIC RESULT, SODIUM
VALPROATE.
THIS SHOWS MORE INFORMATION ON
THAT RESULT.
THIS INCLUDES GRAPH VIEW WHICH
SHOWS ALL THE CONNECTIONS FOUND
BETWEEN POTENTIAL TREATMENT AND
FRAS1 GENE.
YOU CAN SEE THE PATH VIEW
SHOWING ALL PATHWAYS THAT
CONNECT THE TWO IN A TABLE VIEW.
THIS CAN GIVE A GOOD SENSE OF
WHAT KINDS OF RELATIONSHIPS ARE
REPRESENTED IN THE GRAPH ABOVE.
SO IN THIS CASE, FOR EXAMPLE,
VALPROATE CAUSES INCREASED
EXPRESSION OF FRAS1.
IF YOU CLICK ON A GREEN BUTTON,
A SCREEN SHOWS EVIDENCE FOR THIS
RELATIONSHIP.
A SET OF PAPERS FROM PubMed.
THIS SHOWS NOT ONLY INFORMATION
LIKE TITLE AND JOURNAL NAME BUT
ALSO SNIP SNIPPET OF TEXT ON
THE RIGHT SIDE.
THIS WILL TAKE YOU TO THE
PUBLICATION ON THE PubMed
WEBSITE.
WE HAVE OTHER RESOURCES IN
TRANSLATOR AS WELL OF COURSE.
YOU CAN CLICK ON THE SOURCES TAB
AND IT WILL SHOW SOURCES FOR THE
RELATIONSHIP BETWEEN VALPROIC
ACID AND FRAS1, INCLUDING AN
ENTRY IN THE DATABASE.
CLICK ON THE LINK AND YOU'LL
NAVIGATE TO THE CTD HOME PAGE TO
SEARCH FOR FRAS1 AND LINK TO
VALPROIC ACID.
A GOAL IS TO SEND THE USER
DIRECTLY TO THE PAGE WHERE ENTRY
ON THE UNDERLYING SOURCE
DATABASE IF IT EXISTS, IT SHOWS
THE CONNECTION.
THIS IS A LARGE LIFT SINCE
DIFFERENT DATABASES HAVE
DIFFERENT WAYS OF ACCESSING
INFORMATION BUT WE THINK WE CAN
MAKE IT WORK FOR A LARGE NUMBER
OF UNDERLYING SOURCES.
AND THAT PREVIOUS EXAMPLE SHOWED
A STRAIGHTFORWARD GRAPH WITH
FAIRLY DIRECT CONNECTIONS
BETWEEN FRAS1 AND VALPROIC ACID.
ANOTHER SHOWS MORE DEPTH
TRANSLATE TRANSLATOR CAN
PROVIDE.
THIS RECOMMENDS A SET OF
PATHWAYS THAT CONNECT A FORM OF
COLLAGEN ACE.
IF YOU SAY SPECIFICALLY YOU'RE
INTERESTED IN CONNECTIONS
THROUGH COLLAGEN 1A 1 YOU CAN
CLICK ON THAT NODE IN THE GRAPH
TO HIGHLIGHT PATHS THAT INCLUDE
THAT NODE SPECIFICALLY.
YOU COULD CLICK ON THAT NODE A
SECOND TIME TO DESELECT AND GRAY
OUT ANY PATHS THAT GO THROUGH
THAT NODE IF YOU DECIDE IT
WASN'T VALUABLE TO YOUR ANALYSIS
OF THE RESULT.
WE'VE UPDATED COUNCIL IN THE
PAST ON EXCITING OUTCOMES FROM
TRANSLATOR INCLUDING CLINICAL
TRIALS THAT WERE INITIATED FROM
RESEARCH THAT STARTED WITH THE
TRANSLATOR QUERY BUT SOME BEST
STORIES COME FROM THE WORK OF
THE PRECISION MEDICINE INSTITUTE
AT THE UNIVERSITY OF ALABAMA
BIRMINGHAM, PART OF THE
CONSORTIUM SINCE INCEPTION, THE
TEAM WORKS CLOSELY WITH
CLINICIANS WHO SEE PATIENTS WITH
RARE CONDITIONS, USING
TRANSLATOR TO COME UP WITH
POTENTIAL TREATMENTS, WRITE UP
RESEARCH REPORTS AND GIVE
PATIENTS' PHYSICIANS SUGGESTIONS
ALLOWING DOCTORS TO EVALUATE
OPTIONS AND POSSIBLY RECOMMEND
NEW TREATMENT.
OFTEN REPURPOSING A DRUG
INDICATED FOR ANOTHER DISEASE.
EARLY EXAMPLE WAS CYCLIC
VOMITING SYNDROME, LIVES WERE
CHANGED FOR THE BETTER BASED ON
TRANSLATOR RECOMMENDATIONS.
ANOTHER EXAMPLE WAS A RARE
DISORDER DLG 4, SHINE SYNDROME
IS NAMED FOR SYMPTOMS, AS YOU
CAN SEE ON THE SCREEN.
IT'S CAUSED BY MUTATIONS IN DLG
4 GENE THAT CAUSE LOSS OF
FUNCTION OR HAPLOINEFFICIENCY.
RESEARCHERS WERE ASKED TO LOOK
FOR TREATMENTS FOR THIS VERY
RARE SYNDROME.
IT'S NOT FOUND IN UNDERLYING
DATABASES, THEY LOOKED FOR
TREATMENTS THAT MIGHT UPREGULATE
THE GENE SPECIFICALLY TO
COMPENSATE FOR
HAPLOINSUFFICIENCY.
TRANSLATOR RETURNED MANY
POTENTIAL TREATMENTS, A FEW
OPTIONS MAY BE WORTH
CONSIDERING.
BASED ON THIS REPORT AND DOCTORS
REVIEW ONE PATIENT WAS STARTED
ON COMMON BLOOD PRESSURE
TREATMENT, AFTER FIVE MONTHS THE
PATIENT SHOWED MEASURABLE
IMPROVEMENTS IN MOTOR AND
BEHAVIORAL SKILLS.
YOU CAN SEE THAT IN A REMARKABLE
WAY BECAUSE THE PATIENT'S MOTHER
SENT IN IMAGES OF HER COLORING
BOOKS BEFORE HER TREATMENT AND
THEN AFTER TREATMENT.
WHEN SHE REMARKED WE'VE SEEN A
HUGE IMPROVEMENT IN PEPPA PIG, I
REALLY THOUGHT THAT WAS A GREAT
EXAMPLE OF A PARENT
UNDERSTANDING THEIR CHILD'S
ABILITIES IN COMMUNING THEM TO
THE PHYSICIAN IN NON-CLINICAL
TERMS.
WE HAVE MANY OTHER EXAMPLES OF
RESULTS FROM TRANSLATOR QUERIES
LEADING TO ADVANCES AT DIFFERENT
LEVELS.
WE KNOW SEVERAL CASES WHERE
TRANSLATOR RESULTS LED TO
CLINICAL TRIALS INCLUDING
CLINICAL TRIAL FOR TREATMENT OF
ACTIVITY-DEPENDENT NEURAL
PROTECTIVE PROGRAM SYNDROME AS
TRIALS TESTING ACUTE AND LONG
COVID TREATMENTS.
TRANSLATOR HAS DEVELOP FOR EARLY
PRE-CLINICAL RESEARCH ALL THE
WAY TO THE CLINICAL TRIALS
STAGE.
THIS PRESENTATION IS FOCUSED ON
UPDATE ON WHERE WE'VE COME FROM
AND WHERE WE ARE NOW AS FAR AS
TRANSLATOR SYSTEM BUT WE HAVE
IDEAS FOR IMPROVEMENTS, SOME
ONLY POSSIBLE WITH ADDITIONAL
FUNDING PERIOD.
I'LL TALK DURING MY CONCEPT
PRESENTATION ABOUT THIS.
I WOULD BE REMISS NOT TO MENTION
THE RECENT SURGE IN RESEARCH
AROUND LARGE LANGUAGE MODELS
LIKE CHAT GPT ANDOTHERS.
FOR THE MOMENT WE'RE USING ONLY
A FEW TEMPLATED QUERIES IN THE
ALPHA VERSION OF TRANSLATOR ONE
WAY WE HOPE TO EXPAND THIS QUERY
OPTION IS BY ALLOWING USERS TO
ASK A NATURAL LANGUAGE QUESTION,
HAVE AN LLM INTERPRET AND FORMAT
INTO A QUERY THAT TRANSLATOR CAN
UNDERSTAND AND ALSO POTENTIALLY
HAVE PUBLICATIONS SUMMARIZED
ASSOCIATED WITH A GIVEN RESULT
THAT COMES BACK.
TO GIVE USE AREA GOOD OVERALL
IDEA OF TRENDS WITHIN THE PAPERS
THAT ARE ASSOCIATED WITH THOSE
RETURNED RESULTS.
THIS WILL ALLOW FOR THE SYSTEM
TO BE MORE ACCESSIBLE TO
RESEARCHERS AND MUCH MORE
VALUABLE TO THEIR EVERYDAY WORK.
I WANT TO TAKE A MOMENT TO
RECOGNIZE MY COLLEAGUES WORKING
ON TRANSLATOR AT NCATS, TWO
SITTING RIGHT HERE IN THE ROOM,
I MAY THROW QUESTIONS AT THEM,
AS WELL AS THOSE WHO HELPED MAKE
IT POSSIBLE INCLUDING NCATS ITRB
TEAM.
I'D LIKE TO INVITE YOU TO TRY
OUT THE SYSTEM FOR YOURSELF.
WE DO WANT TO CREATE A MOBILE
VERSION OF THE INTERFACE BUT
RIGHT NOW IT'S NOT FORMATTED SO
WE DIDN'T INCLUDE A QR CODE LIKE
JONI LIKES TO DO.
BUT YOU WILL HAVE TO MAKE A USER
ACCOUNT TO RUN YOUR OWN
SEARCHES.
WITHOUT A USER ACCOUNT YOU CAN
SEE RESULTS FROM SEVERAL EXAMPLE
QUERIES INCLUDING RARE DISEASES,
WITH THAT I'D LIKE TO THANK YOU
FOR YOUR ATTENTION.
CHRISTINE AND I ARE HAPPY TO
TAKE QUESTIONS ABOUT THE
TRANSLATOR SYSTEM.
>> CHANGE OF PLANS.
GO RIGHT INTO YOUR CONCEPT.
WE'RE RUNNING REALLY LATE AND I
WANT MAKE SURE THEY CAN WEIGH IN
ON THE CONCEPTS.
>> NO PROBLEM.
ALL RIGHT.
CLEARANCE OF CONCEPTS.
CHRISTINE HAD THE CHANCE TO
INTRODUCE ME, I'LL SKIP PAST
INTRODUCTION SLIDES.
SO THANK YOU FOR ALLOWING US TO
PRESENT THIS CONCEPT.
THIS IS A CONCEPT FOR RENEWAL OF
THE TRANSLATOR PROGRAM.
WE TALK ABOUT TRANSLATOR AND ALL
THE PROGRESS WE'VE MADE.
YOU'VE SEEN THE CURRENT ALPHA
USER INTERFACE HOW RESULTS ARE
DISPLAYED.
WE'LL FOCUS ON THE FUTURE AND
HOW TRANSLATOR CAN BE IMPROVED
UPON AND DEVELOPED INTO A TOOL.
SOURCES COVER A WIDE RANGE OF
NEEDS, DEVELOPED IN A CONTEXTUAL
BUBBLING BASED ON NEEDS OF THE
COMMITTEE THEY WERE BUILT TO
SERVE.
THAT MEANS THEY ARE NOT WELL
ALIGNED TO EXPLORE ACROSS THEM
AND MAKE ALL THE CONNECTIONS
NEEDED.
COMPOUNDING THIS ISSUE IS
RESEARCHERS SOMETIMES AREN'T
AWARE OF RESOURCES WHICH THEY
MIGHT FIND RELEVANT DATA, WE
BUILT TRANSLATOR TO SOLVE THAT
PROBLEM.
IT WAS CONCEIVED TO HELP WITH
DRUG DISCOVERY, STILL PART OF
THE VISION.
OTHER GROUPS WORK ON MODELING
DRUG DISCOVERY AND DRUG
INTERACTION, THESE ARE STILL IN
EARLY STAGES.
INSTEAD WE HAVE TO RELY ON
EXPENSIVE HIGH-THROUGHPUT WET
LAB-BASED SCREENS TO NARROW DONE
THOUSANDS OF POSSIBLE TREATMENTS
BEFORE THEY CAN BE TESTED MORE
THOROUGHLY IN CLINICAL TRIALS.
TO HELP ALLEVIATE ISSUES, TODAY
WE PROPOSE CONCEPT TO
SIGNIFICANTLY IMPROVE UPON THE
CAPABILITIES OF TRANSLATOR
THROUGH ENTERPRISE LEVEL
SOFTWARE ENGINEERING AND DATA
SCIENCE RESEARCH.
I SHOWED THIS TRANSLATOR
TIMELINE SLIDE.
YOU KNOW FEASIBILITY WAS
ACHIEVED IN 2019.
AND WE'RE IN THE DEVELOPMENT
PHASE OF THE PROGRAM.
THE PROGRAM IS NOW RUNNING FOR
FOUR YEARS, WE HAVE ONE YEAR
LEFT OF FUNDING UNTIL THE END OF
THE CURRENT PARADIGM.
WE COMPLETED OUR INITIAL PUBLIC
RELEASE AND SO FAR WE'VE SEEN
OVER 200 USERS BY I.P. ADDRESS.
WHAT I'M ASKING TODAY IS A FIVE
YEAR EXTENSION OF THE TRANSLATOR
DEVELOPMENT PHASE, PERFORMANCE
PHASE.
WE FEEL ANOTHER FIVE YEARS OF
FUNDING CAN GET THIS READY FOR
SUSTAINED FUNCTIONALITY AND
SIGNIFICANT USER GROWTH.
THIS PERIOD WILL CONTINUE TO
INCLUDE TWO TO THREE HACKATHON
TYPE MEETINGS A YEAR WITH
IN-PERSON COMPONENT WHEN
POSSIBLE.
NEXT PHASE WILL BE A FREE AND
OPEN COMPETITION, WE HAVE SOME
IDEAS ON HOW TO CHANGE STRUCTURE
OF THE PROGRAM TO FOCUS EVEN
MORE INTENSELY ON HIGH QUALITY
SOFTWARE DEVELOPMENT AND
IMPROVING RESULTS WE'RE
RETURNING WHILE REDUCING OVERALL
COSTS OF THE PROGRAM YEAR TO
YEAR, CONTINUATION OF USER
INTERFACE CONTRACT.
WE HAVE SEVERAL NEW QUERIES IN
MIND.
WITH BROADER SCOPE THAN ANY
CURRENT QUERIES CAN COVER.
WE WOULD LIKE TO ALLOW USERS TO
DISCOVER CONNECTIONS BETWEEN TWO
CONCEPTS OR CONCEPTS AND ALSO TO
DEFINE CONCEPT AND RELATIONSHIP
TYPE AND FIND ANY OTHER CONCEPTS
THAT FIT THAT RELATIONSHIP.
QUERIES ARE POSSIBLE ON THE BACK
END OF TRANSLATOR BUT IT'S GOING
TO TAKE SERIOUS USER STUDIES TO
DETERMINE HOW BEST TO CREATE
INTERFACE TO ALLOW USERS TO ASK
VERY OPEN-ENDED QUERIES LIKE
THIS.
WE'RE INTERESTED IN EXPLORING
COMBINATION THERAPIES AND IT
COULD BE USEFUL IN THAT REGARD.
WE EXPECT THIS WOULD REQUIRE
EFFORT TO SUCCEED SO CONSIDERED
THIS PART A STRETCH GOAL FOR
THIS PROPOSED FUNDING PERIOD.
WE DO HAVE SOME WORK RELATED TO
THIS BEING DONE BY SOME
TRANSLATOR P.I.s, SO IT'S
WITHIN THE REALM OF THEIR
RESEARCH.
WE HAVE SEVERAL IDEAS FOR NEW
FUNCTIONALITY FOR TRANSLATOR AS
WELL.
WE'VE IMPLEMENTED CREATION
ACCOUNTS, MAKING SURE WE KNOW
WHO IS ACCESSING.
WOULD LIKE TO EXPAND
FUNCTIONALITY TO ALLOW FOR
SEARCH HISTORY AS WELL AS UPLOAD
YOUR OWN DATA TO INTEGRATE WITH
ALL THE OTHER KNOWLEDGE THAT
TRANSLATOR HAS ACCESS TO
POTENTIALLY WITHOUT SHARING DATA
MORE BROADLY, COULD LEAD TO
HAVING A USER WORKSPACE TO DO
MORE USEFUL ANALYSIS AND
EXPANSION OF RESULTS FROM
INITIAL QUERY.
WE THINK THIS IS SOMETHING THAT
DRUG COMPANIES MIGHT FIND
VALUABLE WHICH COULD LEAD TO
EXCITING PARTNERSHIP
OPPORTUNITIES.
IN ADDITION WE'VE BEEN EXPLORING
HOW TO INCLUDE FIGURES RELEVANT
AS PART OF THE EVIDENCE
DISPLAYED.
ONE TEAM MADE PROGRESS.
THERE'S EVEN POSSIBILITY WE
COULD EVENTUALLY EXTRACT
KNOWLEDGE GRAPHS FROM THE
MANUSCRIPT FIGURES TO CREATE NEW
CONNECTIONS HARDER TO FIND
THROUGH TEXT MINING.
WE PLAN TO FOCUS STRONGLY ON
ENTERPRISE LEVEL SOFTWARE
DEVELOPMENT DURING THIS PHASE
AND BY THAT I MEAN WE EXPECT OUR
WORK TO BE UP TO THE STANDARDS
OF MEDIUM-SIZE COMPANY RELYING
ON SOFTWARE TO SUPPORT MANY
CONCURRENT USERS AND STABLE TO
SUSTAIN BOTTOM LINE, WE'LL
IMPROVE DOCUMENTATION ABOUT HOW
SYSTEM OPERATES AND CAN BE
UTILIZED BY USERS, IMPROVE
SCALABILITY TO ALLOW FOR
CONCURRENT USERS, IMPROVE
SECURITY AT ALL LEVELS, AND
UTILIZE INDEPENDENT VERIFICATION
AND VALIDATION SYSTEM TO MAKE
SURE ALL ASPECTS OF TRANSLATOR
ARE OPERATING AT OR ABOVE OUR
STANDARDS.
WE'VE BEGUN THE PROCESS OF THIS
VERIFICATION AND VALIDATION
THROUGH USE OF EXISTING CONTRACT
BETWEEN NCATS AND MITRE, FINDING
ISSUES AND SUGGESTING WAYS TO
FIX THOSE PROBLEMS.
ANOTHER MAJOR COMPONENT WILL BE
BUILDING USER TRUST IN THE
QUALITY OF THE RESULTS THEY SEE
AND COVERING QUERIES MOST
VALUABLE TO THEM.
WE WANT USERS TO KNOW WHERE THE
INFORMATION COMES FROM AND HOW
MUCH IS BASED ON ESTABLISHED
FACT VERSUS EXPERIMENTAL RESULTS
VERSUS MACHINE PREDICTIONS.
FINALLY MAKE IT EASIER TO BUILD
PARTNERSHIP WAS OTHER
ORGANIZATIONS WHO CAN UTILIZE
TRANSLATOR AND CONTRIBUTE MORE
KNOWLEDGE TO THE SYSTEM WHICH
WILL REQUIRE OUR DEVELOPER USERS
TO BE SUPPORTED THROUGH USE OF
APIs AND BACK END SOFTWARE
TOOLS IN ADDITION TO IMPROVED
DOCUMENTATION.
IN AN EFFORT TO GAIN USERS WE'VE
MADE SOME DIRECT LINKS FROM THE
GUARD WEBSITE TO TRANSLATOR AND
EXPECT TO EXPAND THIS
INTEGRATION AND FIND OTHER
SIMILAR PARTNERSHIPS WHERE WE
CAN DIRECT USERS FROM OTHER
KNOWLEDGE HUBS TO TRANSLATOR.
CHRISTINE MENTIONED THE LITCOIN
PROGRAM AND AS IT IS BEING BUILT
RIGHT NOW WE WILL BE ABLE TO
INTEGRATE KNOWLEDGE GRAPHS
GENERATED FROM THAT PROGRAM
SEAMLESSLY INTO TRANSLATOR AS
WELL.
WE EXPECT TO SEE SIGNIFICANT
USER GROWTH WITH THE GOAL OF
ABOUT 2,000 MONTHLY ACTIVE
USERS, SIMILAR TO THE CURRENT
UTILIZATION OF THE MONARCH
INITIATIVES.
AS FAR AS IMPACT WE BELIEVE IN
ORDER TO SUPPORT TRANSLATIONAL
SCIENCE WE NEED TO UNDERSTAND
BIOLOGY AT ALL LEVELS, IF
MOLECULAR TO POPULATION LEVEL.
TRANSLATOR ALLOWS FOR THAT
UNDERSTANDING IN ITS USERS.
WE BELIEVE TRANSLATOR HAS
POTENTIAL TO BE FUNDAMENTALLY
VALUABLE TO RESEARCHERS, SO MUCH
SO WE WOULD LIKE TO SET
AUDACIOUS GOAL OF OUR OWN TO
HAVE TRANSLATOR USED BY RESERVES
AS OFTEN AS PubMed CURRENTLY
IS.
I MENTIONED WE HAVE SOME IDEAS
FOR CHANGING STRUCTURE OF THE
PROGRAM TO TIGHTEN ITS FOCUS AND
REDUCE YEARLY COST OF THE
PROGRAM.
WE'VE UTILIZED OTHER
TRANSACTIONS AUTHORITY FOR
FUNDING RESEARCH PROJECTS AND
CONTRACT SYSTEM FOR BUILDING
TRANSLATOR USER INTERFACE,
PROPOSING TO CONTINUE WITH THIS
OVERALL FUNDING PARADIGM BECAUSE
OF UNIQUENESS OF TRANSLATOR
WITHIN NCATS PORTFOLIO IN SCOPE
AND REQUIREMENTS.
WE PLAN TO CONTINUE USING OTHER
TRANSACTIONS AUTHORITY FOR
RESEARCH TEAMS PART OF
TRANSLATOR.
THIS IS ESSENTIAL FOR THE
FLEXIBILITY OF THE PROGRAM AS
FIELDS OF MACHINE LEARNING
KNOWLEDGE GRAPHS AN LARGE
LANGUAGE MODELS ARE CONSTANTLY
IN A STATE OF FLUX AND CAN CAUSE
UNEXPECTED SHIFTS IN PRIORITIES
THAT NEED TO BE HANDLED AT
PROGRAMMATIC LEVEL.
IN THE PAST YEAR WE ENDED ONE OF
THE TRANSLATOR RESEARCH AWARDS
BECAUSE FOCUS OF THE PROJECT NO
LONGER ALIGNED WITH NEEDS OF THE
OVERALL PROGRAM.
THANKS TO THE OTHER TRANSACTIONS
AUTHORITY FLEXIBILITY WE WERE
ABLE TO REDIRECT FUNDS TO OTHER
RESEARCH TEAMS TO ADD MILESTONES
TO THEIR WORK.
WE HAVE SEEN HAVING 14 SEPARATE
TEAMS CAN LEAD TO SOME
DIFFICULTIES SO WE PLAN TO
REDUCE OVERALL NUMBER OF AWARDS
TO FOCUS TIGHTLY ON PERFORMANCE
AND RESULT QUALITY IMPROVEMENTS.
WE EXPECT THESE WILL BE LARGER
THAN ANY ONE OF OUR CURRENT
AWARDS BUT OVERALL BUDGET WILL
BE REDUCED BY SEVERAL MILLION
DOLLARS YEARLY.
FOR USER INTERFACE WE'VE BEEN
UTILIZING CONTRACT MECHANISM
WITH CONTRACTOR WORKING CLOSELY
WITH RESEARCH TEAMS TO DEVELOP
INTERFACE THAT SUPPORTS NEEDS OF
BOTH USERS AND DEVELOPERS.
THIS IS ALLOWING US TO SET MILE
PHONE IS REQUIRE -- MILESTONE
REQUIREMENTS TIED TO PAYMENT.
WE WOULD LIKE TO EXTEND FOR
TWO-YEAR CONTRACT AS PART OF
THIS CONCEPT.
BECAUSE THIS CONCEPT INCLUDES
CONTRACT PORTION THERE ARE A FEW
TOPICS WE'RE REQUIRED TO
CONSIDER.
SIGNIFICANCE OF THE PROJECT
CAN'T BE OVERSTATED AS WE BUILD
A TOOL THAT GIVES RESEARCHERS A
PLACE TO CONNECT ALL THE
DISPARATE DATA IN THEIR FIELD
SURROUNDING THEIR WORK AND
SUPPORTED DRUG DISCOVERY EFFORTS
IS NCATS PRIORITY.
USER INTERFACE IS EXTANT AND
SUPPORTS QUERIES, AVAILABILITY
OF TECHNOLOGY IS NOT AN ISSUE.
I DEMONSTRATED SEVERAL USE CASES
DURING MY UPDATE THAT SHOWED
CAPABILITIES OF INTERFACE AND WE
EXPECT SIGNIFICANT IMPROVEMENTS
TO BE DONE TO THE INTERFACE
DURING THIS PROPOSED FUNDING
PERIOD.
NOW WE WOULD LOVE TO OPEN THE
CONCEPT TO DISCUSS BY COUNCIL
MEMBERS.
IN SUMMARY WE'RE ASKING NCATS
FUND FOR FIVE YEARS TO IMPROVE
UPON CAPABILITIES THROUGH
SOFTWARE DEVELOPMENT AND DATA
SCIENCE RESEARCH.
RECENT RELEASE OF ALPHA VERSION
SHOWS POTENTIAL AND EXCITEMENT
FOR TRANSLATOR AND WE PLAN TO
IMPLEMENT MAJOR IMPROVEMENTS TO
FUNCTIONALITY AND QUALITY OF
RESULTS DURING THE PROPOSED
FUNDING PERIOD.
I'D LIKE TO OPEN THE CONCEPT FOR
DISCUSSION BY COUNCIL MEMBERS.
I MENTIONED TWO IDEAS THAT WILL
REQUIRE INVESTMENT ON THEIR OWN
BUT COULD BE VALUABLE TO
TRANSLATOR USERS.
FIRST WAS EXPLORING SPACE OF
DRUG COMBINATIONS, USERS TO GET
SUGGESTIONS FOR COMBINATION
THERAPIES FOR SPECIFIC DISEASES
THAT MIGHT BE WORTH ADDITIONAL
RESEARCH.
SECOND IS FOCUSED ON IMAGE
PROCESSING TO EXTRACT KNOWLEDGE
SUCH AS SIGNALING PATHWAYS AND
KNOWLEDGE GRAPH ADJACENT IMAGES.
OUR FIRST QUESTION IS ARE THESE
THE RIGHT STRETCH GOALS?
THE OTHER IS WHETHER THE 2,000
USER GOAL POST, BASED ON CURRENT
UTEIZATION OF MONARCH GRAPHS.
WITH THAT SAID THANK YOU VERY
MUCH FOR YOUR ATTENTION AND I
LOOK FORWARD TO THE DISCUSSION
ON THIS TOPIC.
>> THANK YOU, TYLER.
WE'LL JUST MOVE RIGHT INTO THE
DISCUSSION.
WE HAVE THREE DISCUSSANTS, PAL
HARRIS, MATTIAS, AND ANIKA.
DR. HARRIS?
>> GREAT PRESENTATION.
I LIKE THIS PROJECT AND WHAT
YOU'VE DONE.
COMMENTS AND A RESPONSE.
I THINK IT'S A VERY GOOD GOAL TO
PROFESSIONALIZE THE SOFTWARE AND
CASE HARDEN IT.
YOU SHOULD DO THAT.
BUT I WOULD THINK ABOUT DOING
THAT FIRST WITH THE UNDERLAYER
AND KEEP THE USER EXPERIENCE
PRETTY FLUID AS YOU GO BECAUSE
YOU'LL LEARN A LOT IN THOSE
FIRST USER SETS.
THE OTHER THING ON THE 2,000
MONTHLY USERS, THAT'S THE ONLY
PART THAT SEEMED UNDERBAKED TO
ME, UNDERDEVELOPED.
2,000 USER PER MONTH IS GOOD BUT
I THINK DIGGING DEEPER, HOW MANY
ARE RETURN USERS?
HOW MANY SUCCESSFUL COMPLETIONS?
AND WHAT IS A SUCCESSFUL
COMPLETION IN THIS SPACE?
SPENDING MORE TIME ON YOUR
EVALUATION CRITERIA AND MAYBE
DOING A QUICK LOGIC MODEL WOULD
HELP MAKE SURE YOU'VE GOT THE
RIGHT GOAL IN MIND BEFORE YOU
START.
>> GREAT POINT.
WE LITERALLY JUST LAST WEEK WERE
TALKING WITH USER INTERFACE TEAM
ABOUT IMPLEMENTING GOOGLE
ANALYTICS SO WE CAN GET MUCH
DEEPER INTO WHO THOSE USERS ARE,
WHAT THEY ARE CLICKING ON AND
SPENDING TIME ON, ON THE SITE.
WE'LL DIG IN MORE WHEN THAT'S
IMPLEMENTED ON THEIR END WHICH
SHOULD BE VERY SOON.
>> THANK YOU.
>> YES, REALLY IMPORTANT THAT
YOU KEEP YOUR USER INTERFACE
FLEXIBLE.
UNDERSTAND WITH USERS AND
ASSIGNMENTS YOU CURRENTLY HAVE
TWO, HELPFUL FOR YOU TO BUILD
BASICS FUNCTIONALITIES OUT BUT
POTENTIAL OBVIOUSLY
SIGNIFICANTLY BEYOND THOSE TWO
NARROW USERS.
KEEP YOUR BASE SOLID.
MAKE SURE YOU NICELY ARE
MATCHING INTO THE OTHER CONCEPTS
EXPLODING IN AND ALLOWING YOU TO
USE NEW DATA BUT HAVE GOOD
STRATEGY IN PLACE, WHO USERS ARE
AND HAVE USER CENTRIC DESIGN SO
YOU ARE LOOKING BACK TO THE WAY
YOU MAP YOUR KNOWLEDGE GAPS AND
THIS IS SIGNIFICANT EFFORT.
IT IS INDEED HAS POTENTIAL TO
CHANGE THE WAY WE CONCEIVE GENES
AND DISEASES AND HOW TO BRING
THEM TOGETHER BUT DON'T NARROW
TOO EARLY.
IT'S EASY FOR SOFTWARE TEAMS TO
DO THAT, ALLOWS THEM TO MORE
QUICKLY BUILD SOLUTIONS BUT
YOU'LL CLOSE DOORS WHICH YOU
CANNOT OPEN UP BEGIN.
IF YOU HAVE THAT OPPORTUNITY TO
REACH OUT AS A USER COMMUNITY
AND WHAT NCATS IS DOING AS A
WHOLE WILL HELP YOU TO IDENTIFY
WHAT YOUR USER COMMUNITIES ARE.
THERE ARE THE LOW OF -- A LOT OF
COMMERCIALS, MAYBE ACQUIRE
LICENSES TO SEE WHAT SOLUTIONS
ARE BUILT.
FINAL STATEMENT IS, THIS IS A
STRATEGY DISCUSSION, OPEN
SCIENCE OPEN COMMUNITY EFFORT
MIGHT BE A PLACE WHERE THIS
COULD PLAY OUT VERY NICELY WITH
APIs YOU ALREADY ARE HEADED
THAT WAY BUT IF YOU ESTABLISH A
FRAMEWORK WHERE THEN OPEN SOURCE
COMMUNITIES CAN HOOK TOOLS IN
AND USE YOUR KNOWLEDGE GRAPH FOR
THEIR SPECIFIC SOLUTIONS THEY
ARE BUILDING OUT FOR THEIR USER
COMMUNITIES, SHOULD NOT REPLACE,
BUT THEY WILL BE NOT BE ROBUST,
THEY CAN BE A NICE ADD-ON ON
WHERE YOU STAND.
>> GREAT.
THANK YOU VERY MUCH.
I WILL POINT OUT THAT WE'VE HAD
OPEN SCIENCE AND OPEN ACCESS,
PART OF OUR PLAN FROM DAY 1, AND
WE REALLY TAKE THAT SERIOUSLY.
BUT AT THE SAME TIME WE'RE
ALWAYS LOOKING FOR WAYS WE CAN
PARTNER WITH GROUPS THAT MIGHT
HAVE ACCESS TO DATA THAT WE
DON'T HAVE ACCESS TO NOW THROUGH
THOSE OPEN SOURCES SO THERE'S
KIND OF A PUSH AND PULL THERE,
BUT TRUST ME, RESEARCHERS ARE
VERY STRONGLY, YOU KNOW, INTO
THE OPEN SCIENCE ASPECT OF THIS,
AND I DON'T THINK WE WILL HAVE
ANY TROUBLE MAKING THAT A
PRIORITY MOVING FORWARD EVEN AS
WE'RE GOING DEEPER INTO THE SORT
OF ENTERPRISE LEVEL SOFTWARE
FOCUS.
>> BUT I THINK EXACTLY THIS IS
LAST SENTENCE IS CRITICAL.
YOU BUILD ENTERPRISE SOFTWARE
TOOLS OUT AS RESILIENCE WHO WILL
BE ABLE TO STAND UP AND THEN
HAVE APIs COME IN WHO MIGHT OR
MIGHT NOT HAVE THAT LEVEL OF
SOLUTION.
>> I THINK PART OF THIS
DISCUSSION GETS TO THE
SUSTAINABILITY PIECE AS WELL,
KEEPING IT SOMEWHAT FLEXIBLE,
DIGGING MORE INTO WHO USERS ARE,
PERHAPS INCREASING THAT LEVEL
BECAUSE YOU NEED A LOT OF
DIFFERENT GROUPS, YOU KNOW,
PHYSICIANS, TRANSLATIONAL
SCIENTISTS, EVEN EDUCATION WHERE
THIS COULD BENEFIT, AND OF
COURSE PHARMA.
I THINK BEING ABLE TO UTILIZE
THIS IS POWERFUL.
SO BY HAVING BETTER
UNDERSTANDING OF USERS I THINK
COULD THEN HELP UNDERSTAND WHERE
THIS PRIMARILY MIGHT LAND.
AND THEN THE OTHER PIECE IS
COMMUNICATION, THE TOOL IS
POWERFUL, AND SO YOU HAVE SOME
REALLY GREAT IDEAS FOR HOW TO
DISSEMINATE HOW PEOPLE KNOW
ABOUT IT MORE, KEEPING THAT IN
MIND, AS WELL AS YOU SAY FROM
MOLECULAR LEVEL OR POPULATION
LEVEL PUTTING THE METRICS AROUND
THAT, YOU TALKED ABOUT THE
BENEFITS, REPURPOSING, CLINICAL
TRIALS, EMPHASIZED AS WELL.
>> THANK YOU.
WE ARE JUST STARTING TO GET THE
SYSTEM OUT THERE IN A MORE
PUBLIC WAY.
YOU KNOW, YOU SAW THE LINK.
WE'VE BEEN SHARING THAT MORE
BROADLY.
WE SHALL PROBABLY GOING TO REACH
OUT TO TRAINING PROGRAMS ACROSS
THE COUNTRY BECAUSE WE FEEL LIKE
PEOPLE LIKE, YOU KNOW, EARLY
CAREER INVESTIGATORS, POSTDOCS,
GRADUATE STUDENTS WILL BE EARLY
ADOPTERS OF THIS AND REALLY FIND
VALUE IN IT.
SO I THINK REACHING OUT TO
TRAINING PROGRAMS ACROSS THE
COUNTRY IS A GOOD NEXT STEP FOR
US FOR GETTING THE SYSTEM IN
FRONT OF THE PEOPLE THAT WOULD
ACTUALLY USE IT THE MOST.
>> ANY OTHER QUESTIONS OR
THOUGHTS FROM MEMBERS?
IF NOT --
>> NO FURTHER DISCUSSION I HAVE
A MOTION TO APPROVE THE CONCEPT.
SECOND.
>> SECOND.
>> ALL IN FAVOR?
>> AYE.
>> ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT, CONCEPT IS APPROVED.
THANK YOU VERY MUCH.
>> THANK YOU ALL SO MUCH.
>> I REALIZE WE HAD NO QUESTIONS
FOR CHRISTINE BECAUSE WE MOVED
INTO THE CONCEPT SO IF YOU HAVE
QUESTIONS FOR CHRISTINE, STILL
OPEN FOR THAT.
BUT I THINK WE CAN MOVE ON TO
THE NEXT CONCEPT INTRODUCTION
WITH BALKI AND KARLIE AS WELL.
A JOINT ONE.
>> I'M GOING TO BE BETWEEN THE
PREVIOUS ENTREE AND NEXT ONE.
IT'S LATE IN THE DAY.
I WANT TO QUICKLY LAY FOR YOU
THE FACT THAT THIS IS A CONCEPT
THAT CAN BE FUNDED WITH SMALL
BUSINESS PROGRAMS, THE SMALL
BUSINESS PROGRAMS AT NCATS HAS
127 MILLION, LOTS OF BENEFITS,
WE SUPPORT A LOT OF PROGRAMS
WITHIN NCATS WHICH HAVE
TRANSLATIONAL CONFIDENCE SUCH AS
DIAGNOSTICS, DRUG DISCOVERY AND
DEVELOPMENT, AND THIS CONCEPT IS
GOING TO BE ADDRESSING HEALTH
INEQUITIES IN CLINICAL
DIAGNOSTICS.
KARLIE IS GOING TO PRESENT THIS.
I'M GOING TO STEP AWAY AND HAVE
HER TAKE OVER.
>> THANK, I'M A PROGRAM DIRECTOR
IN THE OFFICE OF DRUG
DEVELOPMENT PARTNERSHIP
PROGRAMS, WE'RE PARTNERING WITH
THE OFFICE OF STRATEGIC
ALLIANCES TO PRESENT THIS NEW
GRANT CONCEPT CLEARANCE TO YOU
TODAY AND IT'S ADDRESSING HEALTH
INEQUITIES IN CLINICAL
DIAGNOSTICS.
OKAY.
SO AS YOU'RE VERY FAMILIAR WITH
THIS, IN ADDITION TO THE VISIBLE
INDICATIONS WE LOOK AT IN
PATIENTS WE USE A WIDE VARIETY
OF LABORATORY ASSAYS AND POINT
OF CARE DEVICES WHEN WE DO
EVALUATION OF PATIENT IN THE
CLINIC.
HOWEVER, THESE ASSAYS UTILIZE
NON-BIOLOGICAL PARAMETERS AS A
PROXY FOR DIVERSITY AND GOOD
EXAMPLE IS SELF-REPORTED RACE
AND ETHNICITY WHICH DOES NOT
NECESSARILY CORRELATE WITH
DIVERSITY OF PATIENT POPULATION.
WE CAN SEE THAT IN THE
PERCENTAGE OF EUROPEAN
CONTRIBUTION TO SEVERAL AFRICAN
AMERICAN SAMPLES WHICH CAN VARY
FROM 3.5% IN SOUTH CAROLINA TO
35% IN SEATTLE.
WE SEE SIGNIFICANT DIFFERENCES
IN GENETIC ANCESTRY OF
INDIVIDUALS WHO SELF-REPORT AS
HISPANIC, MEXICAN AND PUERTO
RICAN INDIVIDUALS SELF-REPORT AS
HISPANIC BUT AS YOU CAN SEE IN
THIS FIGURE THAT DOESN'T
NECESSARILY CORRELATE WITH THEIR
GENETIC BACKGROUND.
WHAT THIS LEADS TO IS THESE
ACASE AND POINT OF CARE DEVICES
OFTEN FAIL MINORITY PATIENT
POPULATIONS, A FEW EXAMPLES, IF
YOU'RE NOT FAMILIAR WITH THE
DRUG THAT INCREASES PLATELET
COUNTS USED IN DISORDERS, BUT
EAST ASIAN PATIENTS RECEIVE HALF
THE DOSE COMPARED WITH NOT, DUE
TO LIMITED PHARMACOKINETIC
STUDIES THAT SUGGEST REDUCED
METABOLISM IN PATIENTS OF EAST
ASIAN DESCENT.
THE RESULT IS SOME PATIENTS
RECEIVE AN APPROPRIATE DOSING,
IN INSTRUMENTS USED TO MEASURE
LUNG FUNCTION, LOWER VALUES OF
FORMAL RANGE FOR AFRICAN
AMERICAN PATIENTS BUT ADJUSTMENT
DELAYS OR PREVENTS DIAGNOSES IN
SOME PATIENTS.
FRACTURE WRIST TOOLS PLACE
AFRICAN AMERICAN WOMEN AT LOWER
RISK OF OSTEOPOROSIS, AND SO
WHAT HAPPENS IN THIS CASE IS
THAT A NUMBER OF HIGH RISK
PATIENTS ARE MISSED, PREVENTIVE
DRUGS ARE NOT ADMINISTERED.
THERE ARE BETTER TOOLS NEEDED
FOR HISPANIC AND LATINO
POPULATIONS.
THERE'S RISK ASSESSMENT
ALGORITHM WITH A
WELL-ESTABLISHED ROLE IN GUIDING
CARDIOVASCULAR DISEASE
MANAGEMENT, HOWEVER THIS
PARTICULAR RISK ASSESSMENT TOOL
AS DESIGNED WAS DESIGNED ON A
PRIMARILY NON-HISPANIC WHITE AND
AFRICAN AMERICAN INDIVIDUAL
PATIENT POPULATION, AND EMERGING
DATA FROM STUDIES INCLUDING
LARGE NUMBERS OF HISPANIC
INDIVIDUALS HAS SUGGESTED THESE
TOOLS ARE NOT ACTUALLY THE BEST
FOR THAT PATIENT POPULATION.
THE FLIP SIDE OF THIS IS THAT
ACTUAL BIOLOGICAL ATTRIBUTES OF
DIVERSE POPULATIONS ARE
OVERLOOKED DURING THE ASSAY AND
DEVICE DEVELOPMENT PROCESS.
INADEQUATE ATTENTION TO BIOLOGIC
CAL DIVERSITY OF PATIENT
POPULATION DURING DEVELOPMENT
PHASE HAS LED TO RELEASE OF END
UTILIZATION OF ASSAYS AND
DEVICES THAT WORK BEST ONLY FOR
SELECT POPULATIONS.
WE SEE THIS IN RACIAL
DIFFERENCES AND DETECTION OF
FEVER.
AS YOU SAW, MANY MEDICAL
DECISION TOOLS DISADVANTAGE
AFRICAN AMERICAN PATIENTS.
IT'S A COMMON KNOWLEDGE THAT
MEDICAL PHOTOGRAPHY HAS FAILED
PATIENTS WITH DARKER SKIN.
AND ON THE RIGHT HERE WE HAVE AN
OPTOMETRY TOOL WHERE RESULTS CAN
VARY.
THE FDA EXAMINED EVIDENCE THAT
PULSE OXIMETERS MAY NOT WORK AS
WELL ON DARK SKIN.
IT'S KNOWN THAT THEY DON'T WORK
QUITE AS WELL WHEN SATURATION OF
OXYGEN IS LOWER THAN 90%, WHAT
THIS DOES IS EXEMPLIFY ISSUE FOR
INDIVIDUALS WHO HAVE DARKER
PIGMENTS.
TESTING AND DEVICES SUFFER,
CAUSING UNDERREPRESENTED GROUPS
TO RECEIVE INFERIOR MEDICAL CARE
AND CAN CAUSE MISDIAGNOSIS.
THESE ISSUES ARE ADDRESSABLE AND
THERE ARE EXAMPLES.
THERE ARE SEVERAL GFR ESTIMATES
THAT MADE SWITCHES TO RACE-FREE
VERSIONS.
THERE ARE PRACTICAL STRATEGIES
FOR IMPROVING CLINICAL
PHOTOGRAPHY OF DARKER SKIN.
WE'VE SEEN A UTI RISK
CALCULATOR, THE MAKER OF THAT
REMOVED THE RACE VARIABLE AND
REPLACED IT WITH TWO OTHER
VARIABLES, LENGTHS OF FEVER AND
PAST HISTORY OF UTI AND HAS NOW
RE-RELEASED THAT PARTICULAR
RESOURCE AND IT IS NOW IN A
RACE-FREE VERSION.
WE SEE SOME WORK OUT OF BROWN
UNIVERSITY ON A SINGLE WAIVE
LENGTH PULSE OXIMETHRY THAT
WORKS ON A LARGER VARIETY OF
PIGMENTS AND CAN AFFECT HOW WE
IMPLANT DEVICES AND DO INTERNAL
IMAGING.
SO WE SEE THAT THIS IS SOMETHING
THAT IS ADDRESSABLE BUT WE DON'T
HAVE A FOCUSED INITIATIVE THAT
ADDRESSES THESE OR PINPOINTS
WHEN THIS IS OCCURRING.
SO INEQUITABLE DEVICES,
LABORATORY ASSAYS, CAN LEAD TO
DELAYS IN CARE OR INACCURATE
DIAGNOSES.
WE WANT TO ADDRESS A GAP FOR ALL
PATIENTS BY PROMOTING
DEVELOPMENT OF IMPROVED ASSAYS
AND DEVICES OR DEVELOPMENT OF
ALTERNATIVE ASSAYS THAT ADDRESS
THIS, SO WE PROPOSE THIS TO
ADDRESS HEALTH INEQUITIES AND
DISPARITIES.
AND INVESTIGATORS THAT COME IN
UNDER THIS PARTICULAR INITIATIVE
WOULD BE EXPECTED TO DEVELOP
DIAGNOSTICS THAT MEET OR EXCEED
QUALITY AND ENCOURAGE ASPECTS OF
SOCIAL DETERMINANTS OF HEALTH.
THIS IS A GOOD FIT WITHIN NCATS
AND OTHER INSTITUTES, AND
CENTERS AT THE NIH.
IN TERMS OF HEALTH DISPARITIES.
IT ADDRESSES A KNOWN PROBLEM IN
HEALTH CARE PROCESSES, WE HAVE
EVIDENCE THAT THE ACADEMIES ARE
WORKING TO MOVE AWAY FROM
NON-BIOLOGICAL PARAMETERS IN
MEDICINE, AMERICAN ACADEMY OF
PEDIATRICS CALLED FOR RACE-FREE
MEDICINE AND THERE'S A
SIGNIFICANT NEED FOR PLAN TO
ADDRESS THESE ISSUES IN ORDER TO
ENSURE THE BEST TREATMENT IS
AVAILABLE FOR ALL PATIENTS.
IT WOULDN'T ONLY INFORM DESIGN
OF SPECIFIC DIAGNOSTICS BUT
WOULD HELP INFORM WHAT IS
WORKABLE FOR ADDRESSING PROBLEMS
AT SALE.
WE EXPECT THIS WOULD IDENTIFY
SEVERAL CLINICAL ASSAYS THAT
FAIL TO INCORPORATE DIVERSE
PATIENT GROUPS AND RELEVANT
NON-BIOLOGICAL PARAMETERS AND
CORRECT TO ENSURE THESE
TREATMENTS ARE AVAILABLE FOR ALL
PATIENTS.
AND SUPPORT WOULD BE PROVIDED TO
SMALL BUSINESSES DEVELOPING
ASSAYS AND DEVICES TO ADDRESS
HEALTH INEQUITIES AND EXPECTED
LONG TERM OUTCOME WOULD BE
RELEASE OF NEWER DEVICES THAT DO
ADDRESS HEALTH INEQUITIES.
WE PROPOSE THIS CONCEPT TO
ADDRESS HEALTH INEQUITIES AND
DISPARITIES IN CLINICAL ASSAY
AND POINT OF CARE SPACE BROADLY
AND WE'RE EXCITED ABOUT THIS
CONCEPT BECAUSE THIS WILL
ADDRESS KNOWN ISSUE IN CLINICAL
DIAGNOSTICS THAT HAS BROAD
IMPACT FOR PATIENT POPULATIONS
AND OUR HOPE THIS WOULD INCREASE
EQUITY AMONGST PATIENTS BY
IMPROVING QUALITY OF CLINICAL
DIAGNOSTICS FOR ALL PATIENTS.
WE HAVE A FEW GENERAL QUESTIONS
FOR COUNCIL MEMBERS.
WHAT MIGHT IMPROVE THIS
INITIATIVE AND WHAT ARE KEY
POINTS TO CONSIDER?
WE HAVE A COUPLE SPECIFIC
QUESTIONS.
ARE THERE PARTICULAR CLINICAL
THINGS WE SHOULD EMPHASIZE SUCH
AS LEVERAGE BIOINFORMATICS OR
GENOMICS?
WE'VE SEEN EMERGING USE OF A.I.
IN THE CLINIC AND CLINICAL
DECISION SUPPORT TOOLS HAS
BECOME MORE PROMINENT, WE WANT
TO KNOW WHAT YOU WOULD SUGGEST
IN TERMS OF CONSIDERING SOFTWARE
AND A.I. TOOLS UNDER THE
INITIATIVE.
WE WOULD WELCOME ANY ADDITIONAL
COMMENTS.
>> ALL RIGHT.
THANK YOU.
WE'LL OPEN UP TO THE PANELISTS
HERE.
GO AHEAD AND PROVIDE SOME
DISCUSSION.
THEY ARE ARGUING OVER WHO IS
GOING FIRST.
GO FOR IT.
>> THIS IS AN EXCITING CONCEPT.
I THINK IT DOES ADDRESS A BIG
GAP THAT CONTINUES THAT WE ARE
RECOGNITION OF BUT NOT ANY
REALLY CONCERTED EFFORTS TO FIND
SOLUTIONS, SO I THINK REALLY
EXCITING TO SEE WHERE THIS GOES
AND I THINK OF COURSE MAKING
SURE, YOU KNOW, THAT THE
GRANTEES ARE ENGAGING DIVERSE
COMMUNITIES AS PART OF THE TEAMS
IS SUPER IMPORTANT.
AS WELL AS HAVING GOOD PLAN FOR
TECHNOLOGY UPTAKE BECAUSE
THERE'S GOING TO BE RESISTANCE
TO CHANGE STANDARD OF CARE.
I THINK COLLABORATION WITH THE
FDA WILL MAKE SURE THAT HAPPENS.
>> YES.
>> THE OTHER THING THAT'S SOUTH
OF SCOPE BUT HEARING EARLIER THE
TALK OF DEIMPLEMENTATION, I
WONDER HOW THIS
WHAT'S CURRENTLY IN USE WE
RECOGNIZE THAT THINGS THAT HAVE
BEEN USED FOR A LONG TIME, IT'S
HARD TO DECOUPLE A LITTLE BIT.
BUT WE DO HAVE PLANS TO INCLUDE
THAT IN THE INITIATIVE, YEAH. T
WEEK.
I DON'T HAVE A TON TO ADD.
PEOPLE ARE GOING TO BE
INTEGRATING A.I. THE CONCEPT IS
BIGGER THAN CLINICAL
DIAGNOSTICS, GETS INTO
MONITORING, AS WE DECENTRALIZE
CLINICAL TRIALS THAT ARE GOING
TO ALLOW PATIENTS TO SELF-SAMPLE
AT HOME, I THINK WE GET -- IT'S
EVEN MORE VITAL, YOUR INITIATIVE
TO DE-RISK TOOLS SO THAT WE
EMPOWER PEOPLE TO PARTICIPATE
AND LET THEM DO IT WITH TOOLS
THAT HAVE BEEN VALIDATED.
I JUST THINK THIS IS AN AWESOME
INITIATIVE.
I LOVE THE LEADERSHIP OF NCATS
TAKING THIS ON TO BENEFIT
RESEARCH ACROSS ALL THE OTHER
INSTITUTES AND ALL OUR DISEASE
AREAS WE CARE ABOUT.
>> THANK YOU.
>> I MIGHT ADD, KARLIE WAS PART
OF ANOTHER INITIATIVE WE DID IN
TERMS OF BIAS IN MACHINE
LEARNING, A.I., CLINICAL CARE.
YOUR POINT ABOUT THIS USING A.I.
TOO IF WE'RE LOOKING
SPECIFICALLY IN UNDERSERVED
POPULATIONS FOR THIS KIND OF
WORK WE MIGHT BE ABLE TO PUT
INSIGHTS WHAT WITH THIS MIGHT
EXIST, COULD THAT BE A SPECIFIC
AREA TO DIVE INTO.
>> THE POINT I HAD BEEN MAKING
BEFORE WHICH BUILDS ON WHAT YOU
WERE SAYING, I THINK IT'S SO
POPULAR TO USE THE WORD A.I.
I CAN'T THINK OF A TOOL THAT I
HAVE VETTED THAT HAS ANY KIND OF
DATA THAT'S PART OF IT THAT
DOESN'T ACTUALLY HAVE AN A.I.
COMPONENT.
DO YOU CALL IT OUT?
AND USE THAT TERM?
OR DO YOU ASSUME THAT'S THE
STATUS QUO?
I'M IN THE CAMP OF NOT USING THE
WORD A.I. EVERYWHERE, BUT I MEAN
IT DOESN'T REALLY MATTER.
IT'S COMING.
IT'S GOING TO BE IN EVERYTHING.
THERE ISN'T ANOTHER DEVICE THAT
ISN'T A DATA COLLECTOR, SO IT'S
WHAT IS THE MEDICAL DEVICE, ALSO
THE SOFTWARE ALSO GENERATING
DATA, A BIGGER QUESTION, HOW YOU
GUYS REFLECT THAT IN THE NOFO IS
IMPORTANT.
AND I HAVE A GUIDANCE QUESTION.
I'M WORKING IN THIS SPACE
EXCLUSIVELY.
DOES THAT MEAN I SHOULD NOT BE
VOTING ON THE APPROVAL?
OKAY.
>> WE WERE DISCUSSING THESE FOR
SEVERAL MONTHS, THOUGHT OF THE
FACT SMALL BUSINESSES
SPECIFICALLY SUITABLE FOR
SOMETHING LIKE THIS, BECAUSE WE
FIND SMALL BUSINESSES TEND TO
HAVE A LOT MORE CAPITAL AND
SOCIAL AWARENESS AND BY DOING
THIS ATTRACT SMALL BUSINESSES
WHICH HAVE GREATER SENSE OF
SERVICE AND MOTIVATION TO MAKE
THIS SUCCESSFUL, A GOOD MARRIAGE
TO HAVE THE SMALL BUSINESS
COMMUNITY APPLY FOR GRANTS AND
CONTRACTS.
>> I'LL JUST SAY ONE SPECIFIC
EXAMPLE TO STRESS HOW IMPORTANT
THAT EMPHASIS IS.
THERE'S A MANUFACTURER BASED IN
L.A.
BODY METRICS.
MAIN TECHNOLOGY IS A WEARABLE
RING, THE FDA JUST APPROVED THEM
AS MOST ACCURATE PULSE OXIMETER
THAT'S A WEARABLE.
THEY HAVE BEEN OFFERED TO BE
BOUGHT BY APPLE.
THEY HAVE BEEN OFFERED TO BE
BOUGHT BY OTHER BIG COMPANIES
BUT THEY KNOW THAT THEIR
TECHNOLOGY WILL NOT ACTUALLY BE
USED FOR THE PURPOSE THEY HAVE
IN MIND WHICH IS TO DO THE RIGHT
THING, AND THEY ARE STRUGGLING
TO STAY ALIVE.
IT'S IMPORTANT TO LET THESE
FLOWERS BLOOM.
THE SMALL BUSINESSES ARE WELL
POSITIONED AND CAN BE MORE
MISSION FOCUSED THAN WHEN THEY
GET ACQUIRED, WE DON'T KNOW
WHAT'S GOING TO HAPPEN TO THE
GOOD IDEAS OF TECHNOLOGY.
YEAH, I HEAR THE PLEAS.
THESE COMPANIES ARE STRUGGLING
TO SURVIVE.
>> GREAT.
GREAT POINTS.
THANK YOU.
OKAY.
>> HEARING NO FURTHER DISCUSSION
MAY I HAVE A MOTION TO APPROVE
THE CONCEPT?
SECOND?
>> SECOND.
>> ALL IN FAVOR?
ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT, OUR LAST CONCEPT OF
THE DAY IS APPROVED.
THANK YOU VERY MUCH.
>> THANK YOU.
>> THANK YOU VERY MUCH.
CHRISTINE IS OFF THE HOOK
COMPLETELY, ANY SPECIFIC
QUESTIONS FOR CHRISTINE TO OPEN
THE FLOOR AND OFFER.
IF NOT, THAT'S OKAY.
IT'S BEEN A LONG DAY.
LONG DAY.
HEARING NONE WE'LL GO AHEAD AND
CONCLUDE FOR THE DAY, AND JUST
AGAIN WANT TO SAY THANK YOU FOR
ALL OF THIS VALUABLE INPUT, WE
TAKE ALL OF THIS INFORMATION TO
HEART AS WE START TO NOW DEVELOP
THE APPROVED CONCEPTS AND MOVE
THEM FORWARD, SO WE'LL MAKE SURE
TO INCORPORATE THE COMMENTS AND
QUESTIONS YOU'VE HAD AND MAKE
THEM REALLY THINK AS ROBUST AS
WE POSSIBLY CAN.
AND WE LOOK FORWARD AGAIN TO
SEEING YOU IN JANUARY, THOUGH IT
WILL BE A VIRTUAL MEETING.
SO WE'LL SEE YOU ON ZOOM.
AND AGAIN THANK YOU, SAFE
TRAVELS ON YOUR WAY HOME.
AND I'LL GAVEL US OUT.
MEETING IS ADJOURNED.