WELCOME TO THE 29TH MEETING OF
THE NCATS ADVISORY COUNCIL AND I
AM GOING TO SWITCH MY WEB CAM
FROM THE GAVEL TO ME SO
HOPEFULLY YOU CAN SEE ME NOW.
EARLIER THIS MORNING WE WERE
HAVING POSSIBILITIES OF SNOW
ICE, THAT SEEMS TO HAVE GONE
AWAY SO I THINK WE WILL BE IN
GOOD SHAPE GOING FORWARD.
THE MEETING TO SPAN A COUPLE OF
DAYS TODAY AND TOMORROW AND WE
HAVE A PACKED AGENDA, HOPE YOU
WILL FIND IT INTERESTING.
BEFORE WE START, ANNA I WILL
TURN IT TO YOU FOR THE OFFICIAL
ROLL CALL.
>> NEXT SLIDE PLEASE.
PAUL HARRIS.
>> HERE.
>> KRISTINA HARTMAN.
KRISTINA, ARE YOU THERE?
TED HOLEMAN.
>> GOOD MORNING.
REBECCA JACKSON.
>> HERE
>> ANNIE KENNEDY.
>> HERE.
>> MATTHIAS KRRETZLER.
>>ER HUE.
>> KELLY MCVEARRY.
>>ER HUE.
>> KEITH MUELLER
>> HERE.
>> RAJESH RANGANATHAN
>> HERE.
PAULA SHIREMAN.
>> HERE.
>> AND MARSHALL SUMMAR.
THOSE ARE OUR COUNCIL MEMBERS.
JONI, BACK TO YOU.
>> THANK YOU SO MUCH.
THIS IS THE AGENDA FOR TODAY'S
PORTION OF THE NCATS COUNCIL AND
WE HAVE A VARIETY AND
PROGRAMMATIC UPDATES AND CONCEPT
CLEARANCES TO I LOOK FORWARD TO
GOING THROUGH THOSE EVENTS WITH
YOU.
I THINK THEN WE'LL ADJOURN FOR
DAY ONE AND COME BACK TOMORROW
AT 1 P.M. EASTERN TIME AGAIN.
AND WE'LL HAVE A VARIETY OF
PRESENTATIONS FOR TOMORROW AS
WELL.
BEFORE WE START WITH DIRECTOR'S
REPORT I'LL TURN IT TO ANNA FOR
ANNOUNCEMENTS TO MAKE SURE WE
ARE ON THE SAME PAGE FOR VARIETY
OF KEY THINGS YOU NEED TO KNOW
ABOUT BEFORE WE START THE DAY.
ANNA BACK TO YOU.
>> THANK YOU, JONI.
EVERYONE IN ZOOM MEETING IS A
PANELIST TODAY SO YOU ARE FREE
TO SPEAK.
HOWEVER IN ORDER TO MAINTAIN A
SMOOTH FLOW WE REQUEST YOU USE
THE RAISED HAND FEATURE AND WAIT
UNTIL CALLED UPON TO PROVIDE
INPUT.
WE ASK YOU RESERVE USE OF THE
CHAT FOR ACTIVITIES SUCH AS
POSTING LINKS PERHAPS,
INFORMATION, BUT PLEASE USE THE
CHAT TO A MINIMUM.
PLEASE ALLOW SPEAKERS TO MOVE
THROUGH THEIR PRESENTATIONS,
NOTING YOUR COMMENTS AND THEN
PRESENTING THEM WHEN CALLED
UPON.
WE ARE USING THE VIDEOCAST
FEATURE TODAY, SO FOR THOSE OF
YOU WHO ARE NOT IN THE ZOOM
MEETING, YOU MAY SUBMIT COMMENTS
BY USING THE EMAIL FUNCTION
AVAILABLE ON THE NIH VIDEOCAST
SITE.
THERE IS A BUTTON THERE AND OR
YOU CAN SEND EMAIL DIRECTLY TO
THE ADDRESS LISTED IN THE SLIDE
NCATS COUNCILINPUT@MAIL.NIH.GOV.
SO THE FIRST THING I WOULD LIKE
TO DO IS CONSIDERATION OF OUR
MINUTES AND OUR NCATS GENERAL
COUNCIL OPERATING PROCEDURES.
WE ARE SEEKING APPROVAL OF THE
MINUTES FOR THE SEPTEMBER 21ST
MEETING, THEY ARE AVAILABLE IN
THE ELECTRONIC COUNCIL BOOK.
COULD WE HAVE A MOTION TO
APPROVE THE MINUTES FOR THE
SEPTEMBER 21ST MEETING?
>> SO MOVE.
>> THANK YOU.
SECOND?
>> SECOND.
>> ALL IN FAVOR?
>> A.
AYE.
>> ANY OPPOSED?
ANY ABSTENTIONS?
THE MINUTES ARE APPROVED.
I WOULD ALSO NOW LIKE TO MOVE TO
SEEKING YOUR APPROVAL FOR
COUNCIL OPERATING PROCEDURES FOR
2022.
THESE ARE ALSO AVAILABLE IN YOUR
ELECTRONIC COUNCIL BOOK.
COULD WE HAVE A MOTION TO
APPROVE THE 2022 COUNCIL
OPERATING PROCEDURES?
>> SO MOVED.
>> HE COULD IS.
>>> SECOND.
>> ALL IN FAVOR.
>> A.
>> ANY OPPOSED?
ANY ABSTENTION?
WITH THAT OUR OPERATING
PROCEDURES FOR 2022 ARE APPROVED
APPROVED.
NEXT SLIDE PLEASE.
FUTURE DATES FOR OUR ADVISORY
COUNCIL MEETING IN 2022 WE PLAN
TO MEET MAY 19 AND SEPTEMBER
22ND FACE TO FACE AS OF NOW.
IN 2023 WE INTEND TO HAVE A
VIRTUAL MEETING ON JANUARY 26
AND 27 AS WE ARE TODAY.
AND TOMORROW AND HAVE FACE TO
FACE MEETINGS ON MAY 25 AND
SEPTEMBER 28TH.
I WOULD LIKE TO POINT OUT TINA
MORRISON FROM THE FDA ALSO
JOINED US TODAY ON BEHALF OF
FRANK WHITEHOLD.
WITH THAT, I WOULD LIKE TO TURN
IT BACK OVER THE JONI.
-- OVER TO JONI.
>> THANK YOU VERY MUCH.
I WILL GET START WITH THE
COUNCIL REPORT AND I HAVE TO SAY
A LOT HAPPENED SINCE WE LAST
MET, SEEMS FOUR MONTHS IS A LONG
TIME.
AND NOT LEAST OF WHICH OF COURSE
IS THAT DELTA HAS COME ON SCENE,
OFF SCENE AND NOW OMICRON IS NOW
THE WE WE ARE DEALING WITH, I
HOPE YOU ARE HANGING IN AND
DOING WELL DURING THIS TIME.
AS I GO THROUGH THE DIRECTOR'S
REPORT PLEASE FEEL FREE TO PUT
QUESTIONS AND COMMENTS IN THE
CHAT AS ANNA MENTIONED, OUR
DEPUTY DIRECTOR CLAIRE SCHMIDT
AND ANN MIGHT BE TAG TEAM WITH
COMMUNICATIONS EDUCATION, THEY
WILL BE MONITORING THE
DISCUSSION AND BE ABLE TO LOOK
AT THE CHAT AND THE RAISED HANDS
AND THEY WILL CALL ON YOU AT THE
END OF THE REPORT AND HOPEFULLY
HAVE GOOD AMOUNT OF TIME FOR
DISCUSSION.
PLEASE DO SO.
FIRST I WANT TO RELAY SOME OF
YOU MAY KNOW, NOT SURE IF
CHRISTINA WILL BE ABLE TO MAKE
IT TODAY, I HOPE SHE IS ON
TODAY, SOME MAY KNOW KRISTINA
HARTMAN IS NO LONGER AT THE
ASSISTANCE FUND.
THIS IS HER LAST OFFICIAL DAY ON
OUR COUNCIL.
SO WE ARE SAYING GOODBYE TO
KRISTINA AND WISH HER WELL IN
HER NEW POSITION AND HER NEW
POSITION PRECLUDES HER SERVICE
ON COUNCIL SO I KNOW THOUGH THAT
WE WILL CONTINUE TO WORK WITH
KRISTINA IN A VARIETY OF OTHER
CAPACITIES.
I KNOW SHE WILL CONTINUE TO
CONTINUE TO GIVE TO THE
SCIENTIFIC IMMUNITY, WE LOOK
FORWARD TO WORKING WITH YOU AND
YOUR NEW ROLE.
>> THANK YOU, JONI.
>> GLAD YOU MADE IT.
NICE TO SEE YOU.
SEVERAL NIH TRANSITION
LEADERSHIP TOO.
DR. COLLINS STEPPED DOWN AS
DIRECTOR OF THE NIH.
HE WAS THE LONGEST SERVING NIH
DIRECTOR AND SERVED THREE
PRESIDENTS.
AND IN CASE YOU MISSED IT, THERE
WAS A WONDERFUL TRIBUTE TO DR.
COLLINS AND THE LINK IS SHOWN
HERE BELOW.
THERE WERE VARIETY OF FAIR WELL
MESSAGES FROM LUMINARY
SCIENTISTS, LIKE JANE GOODALL TO
PRESIDENTS AND VICE PRESIDENTS
AND POLITICIANS TO CO-MEDIANS
AND ACTORS AND MUSICIANS.
SO IT WAS REALLY A WONDERFUL
EVENT SO IF YOU HAVEN'T HAD A
CHANCE PLEASE TAKE A LOOK.
AND AS DR. COLLINS WAS GOING
THROUGH THIS FAIR WELL TOUR HE
ALSO CONDUCTED AN INTERVIEW
PUBLISHED IN JAMA, HE WAS ASKED
ABOUT HIS TOP THREE SCIENTIFIC
ACCOMPLISHMENTS.
HIS RESPONSE INCLUDED THE
FOUNDING OF NCATS AMONG THE TOP
THREE ACCOMPLISHMENTS. I
CERTAINLY COULDN'T AGREE MORE,
COULDN'T BE MORE PROUD OF THAT.
WITH THE VISION OF HIS VISION
FORMING NCATS AND THEN CHRIS
AUSTIN'S LEADERSHIP THE LAST TEN
YEARS IT IS BUILT NCATS FROM
GROUND UP AND NOW IT HAS COME
INTO ITS OWN.
IT IS I THINK EXCITING FOR US TO
BE AND I HOPE YOU ARE AS EXCITED
AS I AM TO CONSIDER THE NEXT
DECADE OF WHAT NCATS IS DOING
FOR TRANSLATION SCIENCE AND RARE
DISEASE RESEARCH AS WELL.
SO I WANTED TO DEFINITELY CALL
THAT OUT FOR YOU TODAY.
THE POSITION OF DIRECTOR IS
PRESIDENTIALLY APPOINTED AND
ALSO SENATE CONFIRMED.
THIS PROCESS IS WHOLLY OWNED BY
THE WHITE HOUSE.
NIH IS NOT INVOLVED AT ALL SO WE
WON'T HAVE ANY EXTRA RUMORS OR
ANY IDEAS FOR THAT MATTER.
NO CANDIDATES TO DATE HAVE BEEN
PUT FORWARD BUT IN THE MEANTIME,
THE PRESIDENT HAS ASKED DR.
LARRY TABAK TO BE ACTING NIH
DIRECTOR AND HE SERVED AS THE
NIH PRINCIPLE DEPUTY DIRECTOR
WITH DR. COLLINS.
ALONG WITH THAT DR. TARA SCHWETZ
HAS TAKEN HIS PLACE AS ACTING
NIH PRINCIPLE DEPUTY DIRECTOR.
MORE TO COME AS WE HEAR NEWS AND
WE WILL HEAR ABOUT IT AT THE
SAME TIME SO STAY TUNED.
OPEN SEARCHES, THE NCATS
DIRECTOR POSITION HAS BEEN
POSTED SINCE WE LAST MET.
THIS ONE DOES NOT REQUIRE A
PRESIDENTIAL APPOINTMENT OR
SENATE CONFIRMATION.
BUT SIMILAR TO THE NIH DIRECTOR
POSITION THIS ONE IS ALSO NOT
WHOLLY OWNED BY NCATS.
THE SEARCH FOR THIS POSITION IS
CONDUCTED BY THE NIH DIRECTORS
OFFICE.
THIS IS THE ANNOUNCEMENT ON THE
SCREEN, IT APPEARED IN A NUMBER
OF VENUES. IT IS OPEN UNTIL THE
END OF THIS MONTH, JANUARY 31ST
AND SO PLEASE HELP SPREAD THE
WORD AND WITH ANY LUCK WE'LL
HAVE AN UPDATE IN MAY I HOPE ON
THIS PROCESS AS WELCH FEW OTHER
NCATS POSITIONS UNDERGOING
TRANSITION THAT I WANT TO TELL
YOU ABOUT.
THE FIRST IS DR. ANTON HE'S BEEN
IN THE ROLE FOR SEVEN YEARS AND
HE DECIDED TO STEP DOWN TO FOCUS
ON OTHER HATS HE WEARS IN THE N
CATS LABS IN THE DIVISION OF
PRE-CLINICAL INNOVATION.
HE'S LOOKING FORWARD TO DEVOTING
THE BULK OF TIME AT CHEMICAL
GENOMICS BRANCH.
WHERE HE'S BRANCH CHIEF OF THAT
MANY THE N CATS LABS.
BUT IN THE MEANTIME ANTON HAS
GRACIOUSLY AGREED TO STAY ON AS
SCIENTIFIC DIRECTOR UNTIL WE
CONCLUDE A SEARCH.
WITH THE NCATS DIRECTOR POSITION
MOVING FORWARD IN PARALLEL WHICH
HOPE TO BEGIN THE NATIONWIDE
SEARCH FOR THE NEXT SCIENTIFIC
DIRECTOR FOLLOWING THE, IN CATS
SEARCH BY A FEW MONTHS.
HOPEFULLY NOT TOO MUCH TIME.
I I'M GRATEFUL WE WILL CONTINUE
TO WORK WITH ANTON IN HIS ROLE
AND HE'S BEEN REALLY A TERRIFIC
PARTNER IN THINKING ABOUT HOW TO
POSITION NCATS LABS GOING
FORWARD.
WE REMAIN IN GOOD HANDS AND
CONGRATULATE ANTON FOR ALL THE
WORK HE CONDUCTED MAKING THE
NCATS LABS WHAT IT IS NOW,
REALLY SUPERMEN SUPER.
I CAN'T BLAME HIM FOR WANTING TO
BACK TO THE LAB EITHER.
WAY TO GO,ANTON.
THANK YOU.
LASTLY, COURTROOM ANN PARISER IS
RETIRING, I ASKED HER TO PRESENT
TO COUNCIL, YOU WILL HEAR HER
PRESENTATION TOMORROW.
I ASKED HER TO PRESENT BECAUSE
THERE IS INCREDIBLE PROGRESS SHE
LED FOR OFFICE OF RARE DISEASE
RESEARCH OVER LAST FIVE YEARS,
SHE CAME TO NCATS FROM THE FDA
TO LEAD THIS OFFICE.
AND SINCE SHE ARRIVED SHE
EXPANDED THE OFFICE SHE'S LED
WORK TO DESCRIBE AND UNDERSTAND
THE BURDEN OF RARE DISEASES.
AND SHE'S DEVELOPED INCREDIBLE
RELATIONSHIPS WITH W IN THE AS
WELL AS ACROSS THE RARE DISEASE
BIOMEDICAL SPACE TO DRIVE ALL
AREAS AND MOST RECENTLY LED THE
WORK FOR NCATS ORPHAN DRUG
STATUS FOR ONE OF THE PAT
TRIALS.
SO WE ARE GOING TO MISS ANN'S
LEADERSHIP AND PASSION FOR RARE
DISEASE RESEARCH SO WE WISH HER
WELL IN RETIREMENT TOO.
DR. PJ BROOKS IS WITH ANN ALONG
THE WAY AND I HAVE ASKED HIM TO
STEP IN AS ACTING DIRECTOR UNTIL
A SEARCH IS COMPLETE.
SO THEY WILL TAG TEAM TOMORROW
FOR THEIR PRESENTATION AS SORT
OF A WARM HAND OFF.
AS WITH THE SCIENTIFIC DIRECTOR
TRANSITION THAT I MENTIONED
BEFORE, THERE WILL BE A SEARCH
FOR NEW DIRECTOR FOR OFFICE OF
RARE DISEASE RESEARCH AS WELL.
THAT WILL COMMENCE RELATIVELY --
AT SIMILAR TIME AS WE CONDUCT
THESE OTHER SEARCHES.
IN THE MEANTIME, WE WELCOME PJ
IN THIS ACTING ROLE AND WE WON'T
SKIP A BEAT WITH HIS LEADERSHIP
THERE.
SO THAT WAS A LOT OF PERSONNEL
CHANGES AND THERE'S A LOT GOING
ON WITH OTHER ANNOUNCEMENTS AND
EVENTS I WILL RELAY.
ALSO VERY EXCITING BECAUSE ONE
IS TO REALLY RECOGNIZE AND
CONGRATULATE OUR LEADERS IN THE
NCATS AND CTSA COMMUNITY.
SHERRY BURKIN ANDREA GARCIA, KEN
MANDL AND LIZ MCNALLY AND KEITH
NORRIS, AND MARCELLA SMITH, IT
UNDERSCORES THE IMPORTANT WORK
WE DO SO CONGRATULATIONS TO ALL
THESE FOLKS.
IN THE SAME VEIN, COLLEEN KELLY,
MEGAN SRINIVAS AND CH ARKANSAS
YANG ARE ALSO RECOGNIZED FOR
THEIR WORK WHAT THEIR ARE DOING
NOW, SOME SCHOLARS AND TRAININGS
AND DR. CHAO YANG IS IN
COMMUNITY ENGAGEMENT, SELECTED
AS PEOPLE, 40 PEOPLE WITHIN
THEIR CATEGORIES WHO ARE UNDER
40.
SO CONGRATULATIONS TO THEM, I
THINK WE ARE DEFINITELY IN GOOD
HANDS GOING FORWARD.
RARE DISEASE DAY IS FEBRUARY 28,
THE LINK AT THE BOTTOM TO
REGISTER IF YOU HAVEN'T DONE SO
ALREADY.
IT IS REALLY A PACKED AGENDA,
ADDRESSING DIVERSITY IN RARE
DISEASE, RESEARCH INEQUITY OF
CARE, IT IS ABOUT PERSONALIZED
MEDICINE IMPORTANCE OF ADVOCACY
AND COLLABORATIONS.
NATURAL HISTORY DATA COLLECTION.
FOR GENE THERAPY TRIALS.
THE USE OF TELEHEALTH AND
EFFORTS ON ADDRESSING THE
DIAGNOSTIC ODYSSEY, THIS IS
REALLY A I THINK A VERY EXCITING
AGENDA WITH MEATY TOPICS REALLY
PRIMING RELEVANT FOR WHAT WE DO
SO HOPE YOU CAN JOIN US I
GUARANTEE YOU THAT THIS WOULD BE
ONE OF THE BEST MEETINGS YOU
ATTEND ALL YEAR SO MAKE IT IF
YOU CAN OR ALERT CUR COLLEAGUES
AS WELL.
SO FINALLY WE HAVE TALKED ABOUT
ARPAH IN PREVIOUS MEETINGS.
AND SO THIS ROUND I DECIDED DR.
SCHWETZ IS NOW BACK TO NIH SO I
ASKED TARA TO JOIN US TO GIVE
UPDATE ON TOPIC OF ARPA H.
SHE'S ACTING PRINCIPAL DEPUTY
DIRECTOR BUT THE LAST SIX MONTHS
SHE'S BEEN DETAILED TO THE
OFFICE OF SCIENCE TECHNOLOGY AND
POLICY AT OSTP.
SHE'S WORKING VERY CLOSELY WITH
DERRICK LANDER ON PLANNING ARPA
H SO I WILL LET HER UPDATE.
I DON'T WANT TO STEAL HER
THUNDER.
SHE WILL FOLLOW ME DIRECTLY.
WAR PA THAT WILL BE THE NEXT TALK.
TO ROUND OUT I HAVE A FEW
UPDATES ON DIVERSITY AND EQUITY
AND INCLUSION OR DEI EFFORT AT
NCATS AND THE NIH.
THE NIH IS HOLDING A SERIES OF
LISTENING SESSIONS AND I HAPPEN
TO HOLD ONE LAST WEEK FOR THE
HEALTH CENTERS AN SYSTEMS, AND
THERE'S ALSO A FEW COMING UP SO
I PUT THOSE DATES HERE SO YOU
CAN SEE THEM BUT THERE IS A LINK
AT THE BOTTOM THAT YOU CAN CHECK
INTO TO SEE OTHER ACTIVITIES
THAT ARE GOING ON IN THIS SPACE
AND REGISTER FOR ONE OF THESE
ACTIVITIES IF YOU ARE ABLE TO.
ENCOURAGE YOU TO JOIN OR SEND TO
COLLEAGUES IF THEY ARE
INTERESTED AS WELL.
IN ADDITION TO THE UNITE EFFORTS
WE ARE DOING AT NCATS WE
ESTABLISHED AN INTERNAL DEIA
HEALTH DISPARITIES GROUP TO
BUILD AND SUSTAIN A WELCOMING
AND INCLUSIVE ENVIRONMENT TO
MAKE SURE THAT OUR PROCESSES AND
OPERATIONS ARE ALL INCLUSIVE OF
THAT HAVE THINKING AND JUST -- I
GUESS IT WAS OVER THE SUMMER
PROBABLY NOW WE ARE ALSO MAKING
SURE THESE ARE IDEAS AND TOPICS
THAT ARE DISCUSSED AT MEETINGS
AND CONFERENCES THAT WE SUPPORT.
ANY TOPICS THAT CAN COVER THE
SCIENCE NEEDS TO ALSO ADDRESS
HEALTH EQUITY IF THERE IS AN
OPPORTUNITY TO DO SO.
WE ENCOURAGE TO HAPPEN IN THEK
SCIENTIFIC OFFICE AS WELL.
ANOTHER CTSA INVESTIGATOR
PUBLISHED A PIECE ABOUT DEI IN
CLINICAL TRANSITIONOR RATIONAL
RESEARCH AND THIS PAPER I DON'T
HAVE A LINK BUT I DID PUT THE
TITLE ON THE SLIDE HERE.
THIS PAPER LAYS OUT A PATH
FORWARD AND IT ALSO REPRESENTS I
THINK A DEEPER EFFORT WITHIN THE
STEERING COMMITTEE AS WELL AS
CTSA COMMUNITY AS LARGE.
THERE IS NOW A DEI TASK FORCE
WITHIN THE STEERING COMMITTEE OF
THE CTSA PROGRAM.
A LOVE OF THE EFFORTS WITH THAT
TASK FORCE BUT ALSO PRIOR TO THE
DEVELOPMENT OF THAT TASK FORCE
THERE'S BEING DISCUSSIONS WITH
CTSA PROGRAM ABOUT THIS.
THIS PARTICULAR PAPER I THINK IS
A CULMINATION OF LOT OF THOSE
DISCUSSIONS THAT TALKS ABOUT
LEADERSHIP AND TRAINING AND
RESEARCH AND CLINICAL TRIALS AND
PROVIDES CONCRETE GOALS THAT
TRANSFORM AND BUILD CAPACITY AS
WELL AS BUILD TRUST.
SO I'M COMMITTED TO TRANSLATING
THESE SORTS OF EFFORTS INTO
MEASURABLE ACTION AS WE GO
FORWARD.
I WANT TO SPEND A FEW SECONDS ON
THE BUDGETS NOT MUCH TO RELAY
SINCE THE LAST MEETING
UNFORTUNATELY.
THE CONTINUING RESOLUTION THAT
WAS TO DECEMBER WAS REUPPED IF
YOU WILL, WE HAVE A CONTINUING
RESOLUTION THROUGH FEBRUARY 18.
JUST REMINDER THAT THE FULL
HOUSE VOTED AND APPROVED THEIR
APPROPRIATIONS BILLS BACK IN
JULY AND THE SENATE -- THEY
COULDN'T AGREE ON THE TOP LINE
APPROPRIATION NUMBERS BUT
MAJORITY RELEASED DRAFT BILLS IN
REPORTS FOR PUBLIC AWARENESS ON
I THINK THAT WAS -- YEAH,
OCTOBER 18.
THESE BILLS HAVEN'T BEEN
FORMALLY INTRODUCED.
WE ARE HOPEFUL OVER THE NEXT
COMING WEEKS WE WILL START TO
SEE THAT MOVEMENT AND FEBRUARY
IS GOING TO BE A CONFUSING
MONTH, NOT ONLY IS THAT
HAPPENING BUT ALSO THE
PRESIDENT'S BUDGET COMES OUT
EVERY FEBRUARY SO THE FY 23
BUDGET IS ANTICIPATED TO BE
RELEASED IN EARLY FEBRUARY AS
WELL.
SO JUST STAY TUNED TO WHAT'S
HAPPENING IN TERMS OF THE
CONTINUING RESOLUTION AS WELL AS
THE FY 22 BUDGET.
I'M ALSO TAKING A MOMENT TO
HIGHLIGHT SOME OF THE RESEARCH
FUNDING OPPORTUNITIES THAT ARE
CURRENTLY ON THE STREET.
I WON'T WALK THROUGH ALL OF
THESE BUT I WANTED YOU TO HAVE
THIS LIST SO YOU WOULD BE ABLE
TO LOOK AT THE DIFFERENT FUNDING
ANNOUNCEMENT OPPORTUNITIES AND
THE TITLES, IT REALLY HIGHLIGHTS
THE BREADTH OF ACTIVITIES THAT
WE ARE INVOLVED IN AND HOPE YOU
AND COLLEAGUES CAN SPREAD THE
WORD OR APPLY TO THESE
ACTIVITIES.
SO I WANT TO MOVE ON TO THE MORE
SCIENCE PORTION OF MY REPORT AND
WILL IS A LOT TO RELAY HERE BUT
WANT TO GIVE A HEADS UP ON OTHER
THINGS THAT WILL BE GIVING YOU
UPDATES ON AS WE GO FORWARD.
VALERIE GORDON WILL TALK ABOUT
INCLUSION MONITORING REPORTS.
THIS IS A REQUIREMENT FOR US TO
RELAY TO YOU ON A, IT IS NOT A
ROUTINE BASIS BUT IT IS A
ROUTINE BASIS BUT NOT FREQUENT
BASIS.
SO SHE WILL TELL YOU ABOUT THAT
REPORT AND WHY IT IS IMPORTANT
THAT WE GET YOUR INPUT ON THAT.
THEN AFTER VALERIE GOES LILY
PORTILLA FROM OFFICE OF
STRATEGIC ALLIANCE WHO WILL
UPDATE ON ACTIVITIES IN HER
OFFICE AND THEN AS I MENTIONED
ANN AND PJ WILL BE TALKING ABOUT
ACTIVITIES AROUND RARE DISEASE
SPACE TOO.
FINALLY KEITH WILL BE TALKING
ABOUT A NEW APPROACH WE ARE TAKE
FOGGER OUR ORGANIZATION OF
NCATS, THAT IS ALSO SOMETHING WE
NEED TO RELAY SO HE WILL TELL
YOU ABOUT THAT MORE TOWARDS THE
END.
NOW I WANT TO DIVE INTO THE
RESEARCH EFFORTS WE HAVE BEEN
DOING.
BEFORE I I SHOULD SAY BECAUSE OF
THE NATURE OF NCATS OUR TEAM
BASED APPROACH, OUR FOCUS ON
PLATFORM TECHNOLOGIES, APPROACH
ON RARE DISEASE AUTOMATION AND
ROBOTICS ACTIVITIES EMPHASIS ON
DATA SCIENCE AND OUR -- ALL HE
IS LEAD TO OUR ABILITY TO
RESPOND TO PUBLIC HEALTH
EMERGENCIES, AND GET INVOLVED IN
VARIETY OF DISEASE RELATED
ACTIVES BUT CERTAINLY OVER THE
LAST COUPLE OF YEARS IT HAS BEEN
FOCUSED ON THIS -- THE COVID
PUBLIC HEALTH EMERGENCY.
ALL OF WHAT WE CAN BRING TO THE
TABLE ARE QUINTESSENTIALLY NCATS
SO I WILL GIVE YOU A SLIVER OF
THE LARGER PIE OF ACTIVITIES WE
DO NOW BECAUSE OF THE COVID
RELATIONSHIP TO THOSE ACTIVITIES
BUT IT DOES JUST REPRESENT A
PIECE SLIVER OF WHAT WE ARE
DOING.
I WILL TELL YOU PROGRESS ON THE
COVID WORK -- COVERING A VARIETY
OF AREAS BUT WILL START WITH THE
CLINICAL TRIALS AND THEN MOVE
INTO THE ANTIVIRAL PROGRAM ON
PANDEMICS AND DIVE INTO OUR
ELECTRONIC HEALTH RECORD
NATIONAL COVID COHORT
COLLABORATIVE ACTIVITIES.
THEN AT THE END OF THE TALK JUST
GIVE YOU A FAIR WARNING, AT THE
END OF THE TALK I WANT TO SWITCH
GEARS THE LAST TEN OR SO MINUTES
TO WALK THROUGH SOME HIGH LEVEL
GOALS AND I WANT TO TACKLE OVER
THE NEXT DECADE AND WITH WHAT I
PRESENT TODAY I HOPE IT SERVE AS
BROAD RANGE OF SCIENTIFIC
PURSUIT AND THE GOALS OUTLINED
EMBRACE THAT APPROACH AND WILL
SERVE TO HELP DRIVE THAT SUCCESS
AS WE GO FORWARD.
SO FIRST UP OF COURSE IS THE --
I MENTION CLINICAL TRIALS.
THE CTSA PROGRAM AGAIN HAS BEEN
DOING THE LION'S SHARE OF THE
COVID CLINICAL TRIAL WORK
THROUGH THE ACTIVE PROGRAM THAT
WE HAVE THROUGH THE FNIH AS WELL
AS THE NIH AND VARIETY OF
PRIVATE PARTNERS.
WITH THIS PUBLIC PRIVATE PARTNER
FRAMEWORK WE HAVE -- WE HAVE
DONE A VARIETY OF CLINICAL
TRIALS AND SOME OF THEM HAVE
BEEN NCATS WHOLLY SUPPORTED LIKE
THE CONVALESCENT PLASMA TRIALS
BUT SOME THROUGH THE PUBLIC
PRIVATE PARTNERSHIP THAT NCATS
HAS SPONSORED.
MOST MONEY COMING ARE NOT NCATS
DOLLARS, THESE ARE DOLLARS
(INAUDIBLE) RESPONSE.
I'M GOING TO TALK ABOUT THE
CONVALESCENT PLASMA TRIALS,
THERE'S EARLY WORK COMING OUT, A
BIGGER STORY TO TELL THAT WE
WILL HEAR MORE OVER THE NEXT
COMING MONTHS BUT THERE'S ONE
REPORT OUT THAT I CAN TALK ABOUT
RELATED TO THE CONTAIN TRIAL.
I'M TALKING ABOUT SOME OF THE
OTHER ACTIVITIES TOO.
TESTIFY CONTAIN STUDY IS LOOKING
AT EFFICACY OF COVID-19
CONVALESCENT PLASMA IN
HOSPITALIZED PATIENTS.
THIS IS OUT OF THE NEW YORK
UNIVERSITY AND CTSA HUBS AND
AFFILIATES WITH THEM.
AND THE TOP LINE IS THAT
CONVALESCENT PLASMA DID NOT MEET
THE PRE-SPECIFIED CRITERIA FOR
EFFICACY IN THIS STUDY.
THEY WERE USING THE HUE SCORE
FOR SEVERITY, 11 POINT SCALE
WHERE 0 IS ASYMPTOMATIC AND 10
IS DEATH.
AND THIS GRAPH HERE SHOWS
THERE'S ODDS RATIOS POST TIER
YEAR PROBABILITY ANALYSIS SO
THAT RED LINE THERE IN THE
MIDDLE IF YOU ARE TO THE LEFT OF
THAT LINE THAT SHOWS A LITTLE
BIT MORE BENEFIT, BUT FOR THIS
THERE'S NO SIGNIFICANT BENEFIT
THAT IS INDICATED IN GENERAL
WITH CONVALESCENT PLASMA.
BUT WHAT WAS INTERESTING IS WHAT
THEY FOUND WHEN THEY DID
SUBGROUP ANALYSIS IS THAT EARLY
IN THE PANDEMIC WHEN THERE WERE
NO TREATMENTS, IT SHOWED SOME
EFFECTIVENESS IN THAT SUBGROUP
ANALYSIS.
HOWEVER, AS REMDESIVIR AND
CORTICAL STEROIDS AND OTHER
TYPES OF TREATMENT VACCINES ARE
ON THE SCENE, IT HAS -- IT HAS
WANED IN THAT BENEFIT SO THERE'S
NO LONGER REALLY A BENEFIT FOR
CONVALESCENT PLASMA THAT WE ARE
SEEING NOW.
THE CONVALESCENT PLASMA STORY
ISN'T DONE YET SO KEEP YOUR EYE
OUT FOR OTHER REPORTS COMING
SOON FROM THE WORK WE SUPPORTED
AS WELL.
AS WELL AS OTHERS.
ACTIVE ONE ENDED ENROLLMENT WITH
THE LAST PATIENT FOLLOW-UPS THAT
WILL HAPPEN IN FEBRUARY AND
MARCH.
THIS ACTIVE ONE STUDY LED BY
BILL WASHINGTON UNIVERSITY ST.
LOUIS CTSA ALONG WITH 30 OTHER
CTSA SITES INVOLVED WITH OTHER
SITES TOO.
THIS IS A DOUBLE BLIND PLACEBO
CONTROL TRIAL AND HOSPITALIZED
PATIENTS WITH RANDOMIZED ACROSS
THREE DIFFERENT
IMMUNOMODULATORS.
SENATE P -- SENEPIVEROC ENDED
EARLY BUT TOP LINE RESULTS DID
COMPLETE ENROLLMENT AND WE WILL
BE HEARING MORE ABOUT THESE
AROUND MAY OR SO WHEN WE GOT TO
GET THAT DATA AND ANALYSIS
COMPLETED.
STAY TUNED IN MAY, HOPEFULLY
WE'LL UPDATE THEN.
ACTIVE 6 IS LED BY DUKE AND DCRI
AS WELL AS VANDERBILT AS
COORDINATING CENTER.
IT IS AN OUTPATIENT TRIAL TO
TEST EXISTING DRUGS APPROVED FOR
OTHER INDICATIONS TO SEE IF THEY
WERE BE REPURPOSED FOR COVID-19
SYMPTOM RELIEF.
THIS PARTICULAR CLINICAL TRIAL
IS LOOKING AT THREE MAIN DRUGS,
IVERMECTIN AND FLUVOXAMINE.
WE ARE SENDING THESE MEDICATIONS
TO PATIENT HOME AND DO FOLLOW-UP
ONLINE OR THROUGH TELEPHONE
SURVEYS SO IT IS A UNIQUE TRIAL
WITHIN THE ACTIVE PARTNERSHIP.
BUT IT IS GOING VERY WELL AND
TARGETED ENROLLMENT SHOULD BE
MET BY END OF MAY, SORRY BY END
OF I GUESS I'LL SAY SPRING,
PROBABLY MARCH, APRIL MORE
LIKELY.
AND HOPEFULLY WE'LL BE ABLE TO
GIVE MORE DETAILS ON THIS
ACTIVITY IN OUR MAY COUNCIL.
WE ARE ALSO RAMPING UP OUR
ANTIVIRAL PROGRAM FOR PANDEMICS.
THIS IS A COLLABORATION WITH M
CATS, NIAID, AND BARTA AND THIS
IS TO CREATE PHASE 2 TRIAL READY
ANTIVIRALS FOR RAPID PANDEMIC
RESPONSE SO THEY WILL GET
THROUGH CERTAINLY AT LEAST PHASE
2 AND BE READY OR GET THROUGH
PHASE 1 AND BE READY FOR PHASE 2
IN CASE THERE IS ANOTHER NEED
FOR A PANDEMIC RESPONSE.
WE HAVE DONE SURVEY OF VIRAL
FAMILIES AND ESSENTIALLY SEVEN
VIRAL FAMILIES THOUGHT TO BE OF
THE HIGHEST FOR PANDEMIC
POTENTIAL.
SO THOSE ARE SHOWN HERE ON THE
LEFT.
GIVEN WE ARE CURRENTLY IN THE
PANDEMIC OF THE CORONA VIRUS
FAMILY, MOST OF THE WORK WE DO
CURRENTLY IS RELATED TO CORONA
VIRUS.
SOME OF THE PROJECTS DOING TO
DATE, ARE LISTED HERE WITH CLEAR
CREEK BIO COLUMBIA UNIVERSITY
AND NCATS SUPPORTING THESE
ACTIVITIES AS WELL.
WE ARE LOOKING AT THINGS LIKE
PROTEASE INHIBITORS TO STOP
VIRAL REPLICATION WAY TO STOP
VIRAL REPLICATION AND
INFECTIVITY.
WE ARE ALSO LOOKING AT COUPLE OF
ANALOGS RNA DEPENDENT RNA
POLYMERASES, AND THESE SORTS OF
INHIBITORS TO ALSO HELP PREVENT
VIRAL REPLICATION.
SO THESE ARE SOME OF THE
PROJECTS IN THE WAY IN OUR NCATS
LABS IS LEADING THIS EFFORT FOR
NCATS.
WE HAVE A GREAT TEAM PUSHING
REALLY A LOT OF HE IS SORTS OF
ACTIVITIES -- THESE SORTS OF
ACTIVITIES AND WE ARE OPEN FOR
BUSINESS SO ANY IDEAS ALONG THE
WAY, WE ARE HAPPY TO HEAR ABOUT
THEM.
EXCITING STUFF AS THIS IS
GETTING OFF THE GROUND.
SO I WANT TO ALSO JUST SHOW YOU
SOME OTHER ACTIVITIES THAT WE
ARE DOING IN TERMS OF MAKING
SURE DATA ARE READILY ACCESSIBLE
AND AVAILABLE FOR SCIENTISTS AND
RESEARCHERS AND OTHERS.
WE ARE LEADING THIS EFFORT WITH
THE ACTIVE GROUPS TO FOLLOW ALL
THE ACTIVE RELATED ACTIVITIES
AND VACCINES AND THERAPEUTICS.
AND THIS IS SORT OF A ONE PLACE
WE CAN GO TO EASY TO READ DATA
IN THIS OPEN DATA PORTAL.
IT IS ON OUR NCATS WEBSITE.
IT IS -- SO JUST TO ORIENT YOU
AT THE TOP CORNER WITH ALL THOSE
LITTLE DOTS THAT ARE DIFFERENT
COLORS THOSE REPRESENT THE
DIFFERENT WAVES OF THE DIFFERENT
VARIANTS OF THE VIRUS.
ON THE LEFT-HAND SIDE, ALL THAT
IS REALLY REPRESENTING JUST THE
OMICRON WAVE DATA.
SO ALL THE DATA WE COLLECTED AS
OMICRON HAS COME ON TO SCENE AS
RELATES TO THE VACCINES AND THE
DIFFERENT MOAN:AL ANTIBODIES AS
WELL AS OTHER THERAPEUTICS.
SO ALL THOSE DOTS THERE ON THE
RIGHT HAND SIDE INDICATE THAT
ACTIVITY IS WANED BUT IF THE DOT
IS COLORED ON THE LEFT-HAND
SIDE, THAT MEANS NO REDUCTION IN
BENEFIT AND THOSE ARE ACTUALLY
VIABLE CANDIDATES FOR
THERAPEUTICS TO HELP THE OMICRON
PIECE OF THE PANDEMIC.
ON THE RIGHT HAND SIDE SHOWS THE
DIFFERENT KINDS OF ASSAYS THAT
WE CONDUCT TO SHOW THIS
VIABILITY EFFICACY, THESE TYPES
OF MEDICATIONS.
SO IT IS REALLY A GREAT RESOURCE
TO LOOK, NOT JUST AT CURRENT
RATE BUT ALSO THE OTHER WAVES TO
SEE THE DIFFERENCES AND WHY IT
IS VERY IMPORTANT WE HAVE THIS
LARGE THERAPEUTICS PROGRAM, THAT
IS HELPFUL AS WE MOVE FORWARD IN
THIS PANDEMIC WHICH IT IS -- I
DON'T KNOW THAT ANY OF US COULD
HAVE PREDICTED IT WOULD BE HERE
TODAY WHERE WE ARE SO I HOPE WE
CAN CONTINUE TO MAKE THESE
THINGS AVAILABLE AND CONTINUE
THE ACTIVITIES GOING FORWARD.
THE OTHER MAJOR AREA IS AS I
TALKED BEFORE IS MAKING
ELECTRONIC HEALTH RECORD
INFORMATION AVAILABLE FOR
STUDYING COVID-19.
THIS IS A PARTNERSHIP WITH THE
SAVE PROGRAM 2H ALONG WITH
NATIONAL INSTITUTE ON GENERAL
MEDICAL SCIENCES AND THE IDEA
CLINICAL RESEARCH PROGRAM AS
WELL AS NON-PROFIT HEALTH IT
ORGANIZATION WORKING TO IMPROVE
HEALTH EQUITY.
NCATS IS JOINED UP WITH OTHER
FEDERAL AGENCIES AS WELL FOR
EXAMPLE THE FDA, OFFICE OF
NATIONAL COORDINATOR FOR HEALTH
INFORMATION AND TECHNOLOGY AS
WELL AS BRTA AND A FEW OTHERS.
TO DATE, THERE ARE NOW OVER 10
MILLION ELECTRONIC HEALTH
RECORDS WITHIN THE DATA SET WITH
3.7 MILLION REPRESENTED COVID
POSITIVE CASES.
OVER 12 BILLION ROWS OF DATA
WITH ENTIRE COMMUNITY WHO IS
MAKING HEADWAY ON ORGANIZING AND
ANALYZING THIS DATA.
AND THERE IS A DASHBOARD HERE AT
THE BOTTOM, A LINK THERE YOU CAN
GO TO NCATS DASHBOARD AND YOU
CAN SEE THIS DATA COLLECTED.
BUT AS I MENTION 12 BILLION ROWS
OF DATA SO THINK ABOUT THAT ROWS
OF DATA, AND IF YOU RUN THE
SPREADSHEET AND PRINT OUT THAT
SPREADSHEET AND YOU THINK ABOUT
EACH CELL IN A ROW IS PROBABLY
AN INCH, THE AMOUNT OF DATA THAT
IS BEING COLLECTED, IF YOU WERE
TO TAKE THE PREED SHEET AND MAKE
LINEAR ACROSS EACH ROW, THAT ROW
WOULD GO AROUND THE EARTH MORE
THAN THREE TIMES.
SO I AM GLAD WE ARE DIGITAL, IT
IS VERY HELPFUL BUT IT IS A LOT
OF DATA BEING CHECKED AND REALLY
IMPRESSIVE WORK THAT THIS GROUP
HAS DONE.
WE HAVE DONE BIT MORE TO AUGMENT
DATA AS YOU MIGHT IMAGINE,
ELECTRONIC HEALTH RECORDS CAN BE
MESSY AND INCOMPLETE SO WE WANT
TO MAKE SURE WE ARE DOING OUR
DUE DILIGENCE TO GET AS MUCH
INFORMATION AS POSSIBLE.
SO WE HAVE THE DATA HERE IS NOT
REALLY THAT CRITICAL BUT IT IS
NOW INCLUDING MORTALITY DATA, IT
IS INCLUDING VIRAL VARIANT
SEQUENCE DATA SO WE KNOW WHICH
VARIANTS PARTICULAR PEOPLE HAVE
BEEN EXPOSED TO AND HOW THAT
MIGHT DRIVE OUTCOMES.
WE ARE GETTING CMS DATA WHICH
INCLUDES CLAIMS DATA,
PRESCRIPTIONS AND OTHER
INDICATORS OF HEALTHCARE
UTILIZATION.
SO THESE ADDED ON DATA SETS WILL
BE MADE AVAILABLE LIKELY
STARTING IN BEGINNING OF
FEBRUARY.
AND WE ARE NOT DONE THERE, WE
WANT TO GO AND THINK ABOUT WHAT
IS NEXT SO WORKING TOWARD
FIGURING WAYS OF OBTAINING ICU
WAGE INFORMATION FORM AS WELL AS
IMAGING DATA.
SO WE HAVE REALLY MOVED THE BAR
IN ADOPTING PRIVACY TECHNIQUESES
TO ENABLE THIS TYPE OF ACTIVITY
AND MAXIMIZE POTENTIAL OF THESE
EXTERNAL DATA SETS AS WELL AS
OTHER FUNDED REPOSITORIES LIKE
THOSE BEING DEALT FOR RECOVER
PROGRAM THAT POST ACUTE SEQUELAE
OF COVID CONDUCTED OUT OF NHLBI
WE ARE PARTNERING WITH THEM SO A
VARIETY OF THINGS PULLED INTO
AND I THINK THE IS MAKING THIS
DATA SYSTEM VERY ROBUST.
I WILL GIVE YOU A SENSE OF
ACTIVITIES ONGOING IN A MOMENT
BUT I WANT TO RELAY TWO MORE
POINTS THAT ARE NEW RELATED TO
THAT.
ONE IS WORKING TOWARDS A TRIBAL
CONSULTATION, WE HAVE BEEN
WORKING THIS OVER A YEAR BUT THE
REASON THAT IT TOOK A BIT TO GET
THERE, THERE'S OTHER ACTIVITIES
AROUND COVID THAT ARE ALSO
TRIBAL CONSULTATION SO WE HAVE
BEEN IN THE QUEUE AND IT IS NOW
OUR TURN.
THE REASON THIS IS IMPORTANT,
WHEN WE GET TO DEMOGRAPHIC FROM
DATA CONTRIBUTORS IT CONTAINS
THE OMB RACE TABLES WHICH
INCLUDES AMERICAN INDIAN ALASKA
NATIVE OR AIAN DATA.
THESE ARE SELF-IDENTIFIED OR
PROVIDER IDENTIFIED
CLASSIFICATION OF RACE FROM
EXISTING DATA.
WE DON'T RECEIVE TRIBAL
AFFILIATION DATA, SIMPLY THE
RACE TABLES WE GET.
KEEPING INTEGRITY OF THE TRIBAL
SOVEREIGNTY AND PRIVACY WHILE WE
ARE WAITING TRIBAL CONSULTATION
WE HAVE BEEN MEETING WITH OUR
TRIBAL HEALTH RESEARCH OFFICE
AND CENTERS ACROSS THE COUNTRY
AND OTHER EXPERIENCED NIH POO TO
DISCUSS OPTIONS WHAT WE CAN DO
UNTIL WE HAVE THAT CONSULTATION.
BASED ON DIVISION CUSHIONS WITH
THE TRIBAL RESEARCH OFFICE,
NCATS DECIDED AT THAT TIME
OUTSET WHEN WE FIRST MADE THE
DATA PUBLICLY AVAILABLE WE
OBSCURED THE AIAN RACE DATA AS
WELL AS OBSCURED THE 2,659 ZIP
CODE DATA THAT OVERLAPS WITH
TRIBAL LANDS TO PREVENT
INFERENCE OF TRIBAL AFFILIATION.
THESE ARE TWO APPROACHES WE CAN
DO UNTIL WE HAVE TRIBAL
CONSULTATION.
WE LOOK FORWARD TO MEETING WITH
TRIBAL LEADERS AND HAVE AN
OPPORTUNITY TO PROVIDE WHETHER
AND HOW TO UTILIZE THESE SORTS
OF DATA AND (INAUDIBLE) WHICH
MIGHT BENEFIT THE COMMUNITY AS
WELL.
IN FEBRUARY WE WILL BE HAVING
THIS TRIBAL CONSULTATION AND
VARIETY OF ACTIVITIES WILL
FOLLOW SUIT.
THE SECOND AND LAST UPDATE IS
REALLY GIVE YOU A I WANT TO SHOW
AN OPPORTUNITY THAT CAME ACROSS
WITH US, THIS IS A REAL WORLD
EVIDENCE STUDY OUT OF UNIVERSITY
OF COLORADO BY THE INDIANS --
THEY ARE LOOKING AT
EFFECTIVENESS OF TREATMENT WITH
MONOCLONAL ANTIBODIES.
THERE IS AN OPPORTUNITY HERE TO
LOOK AT THE IMPACT OF VIRAL
VARIANTS SPECIFICALLY ON
MONOCLONAL ANTIBODY
INTERVENTION.
SO THIS IDEA CAME ACROSS BECAUSE
OF DISCUSSION WE HAD WITH JANET
WOOD COKE WHCOKC, ABOUTING DEPUTY
COMMISSIONER OF THE FDA BUT ALSO
WHITE HOUSE LEADING A LOT OF
ACTIVITIES RELATED TO THE COVID
RESPONSE.
SO THIS CAME OUT OF JANET'S HEAD
AND WE WANTED TO SEE THIS KIND
OF STUDY HAPPEN.
AND BECAUSE OF THE OPPORTUNITIES
WE HAVE WITHIN NCATS THERE WAS A
WAY TO GET GET COVID SPECIFIC
FUNDS TO SUPPORT THIS KIND OF
ACTIVITY.
NOW BEHOLD THIS PROJECT IS
ESSENTIALLY TO ENROLL 4,000
INDIVIDUALS SOME WHO RECEIVED
MONOCLONAL ANTIBODIES AND SOME
WHO DIDN'T AND FOLLOW THE
INDIVIDUALS WITH TELEPHONE
SURVEYS AND LIKE, WE ALSO
RECEIVE ELECTRONIC HEALTH RECORD
INFORMATION TOO.
AND THIS IS A WAY FOR US TO
DETERMINE THE EFFECTIVENESS OF
THE TREATMENT AND PREVENTING
HOSPITALIZATION AND ATTRACT --
TO TRACK THOSE LONGER TERM
SYMPTOMS AS WELL.
SO THE UNIVERSITY OF COLORADO
TEAM RECOGNIZED THAT WE REALLY
NEEDED TO UNDERSTAND WHO IS MORE
AT RISK TO GET THE DISEASE AND
MORE AT RISK TO BE HOSPITALIZED
SO THAT THE LIMITED SUPPLY
PERHAPS SOMETIMES OF THESE
MONOCLONAL ANTIBODIES COULD BE
USED AND DIRECTED EFFICIENTLY
AND MOST EQUITABLY.
SO THESE ARE THE DATA THAT I'M
SHOWING HERE ON THE SCREEN, IT
IS THIS IS OUT OF NET ARCHIVE,
NOT PUBLISHED YET BUT IT IS
PRE-PRINT SERVER ON MED
ARCHIVES.
SO STILL ONGOING BUT EARLIER
RESULTS THESE ARE DATA THROUGH
JULY SHOW IMPRESSIVE RESULTS BY
DAY 28 FOR EXAMPLE, THIS FIRST
RED LINE HERE, THE MONOCLONAL
ANTIBODY SHOW 50% EFFECTIVENESS
PREVENTING HOSPITALIZATION AND
THEN DOWN AT BOTTOM YOU SEE IT
IS QUITE EFFECTIVE IN TERMS OF
THE LENGTH OF STAY AND
MINIMIZING LENGTH OF STAY OF
THOSE WHO ARE HOSPITALIZED.
THEN THERE'S ALSO BY DAY 90 IN
TERMS OF ALL CAUSE MORTALITY,
THERE'S 83% EFFECTIVENESS IN
PREVENTING DEATH.
SO THESE ARE REALLY IMPORTANT
DATA FOR US TO UNDERSTAND AND
KNOW AND FIGURE HOW TO BEST USE
THESE KINDS OF TREATMENTS IF
THEY ARE IN LIMITED SUPPLY.
SO WE LOOK FORWARD TO I HOPE
BEING ABLE TO EXPAND SOME OF
THIS WORK, AND ESPECIALLY AS THE
PANDEMIC CONTINUES, THIS KIND OF
INFORMATION IS VERY VALUABLE.
THIS IS A LOT MORE WORK COMING
OUT OF N 3C, HIGHLIGHT A FEW
PAPERS HERE IN TERMS OF
PUBLICATION AS WELL AS PRE-PRINT
AND SOME OF THE NEWS OUTLETS
CAPTURING A LOT OF THE WORK
GOING ON IN NTC SO THAT'S
EXCITING TO SEE THIS UPTAKE OF
THIS PARTICULAR DATA SET, IT IS
QUITE RICH AND IT IS GETTING
RICHER WITH THAT INFORMATION I
KNOW IT WILL BE VALUABLE.
WITH THAT ADDED INFORMATION.
IT IS HARD SOMETIMES GET
MAGNITUDE ASIDE FROM THE FACT
THE DATA GO AROUND EARTH THREE
TIMES.
LILY PORTILLA SENT ME NUMBERS OF
AGREEMENTS IN SUPPORT OF N 3C
THAT WE OBTAIN TO DATE, IT IS
AMAZING, THIS IS TRULY AN
ENTERPRISE LEVEL COLLABORATION
WITH OVER 400 PAYMENTS IN PLACE
THAT SUPPORT THIS WORK.
SO I JUST GIVE YOU A SENSE OF
THE MAGNITUDE OF THAT.
SO I'M GOING TO SWITCH GEARS A
LITTLE BIT NOW, I WANT TO CLOSE
OUT BY TALKING ABOUT WHAT IS
NEXT FOR NCATS.
ON DECEMBER 7 AS YOU MAY HAVE
REMEMBERED IT IS OUR BIRTHDAY,
AND WE HAD OUR BIRTHDAY PARTY
AND IT WAS JUST REALLY A
WONDERFUL EVENT.
WE HAD OUR 10TH ANNIVERSARY
CELEBRATION WHICH WHERE WE HAD
771 ATTENDEES AND AVAILABLE ON
DEMAND, YOU ARE WELCOME TO VIEW
ON DEMAND AS WELL.
BUT IT GAVE US AN OPPORTUNITY TO
TAKE STOCK AND TALK ABOUT MANY
OF THE PROGRAMS AND TOPICS THAT
ARE LISTED HERE FROM PATIENT
IMPACT TO DATA DRIVEN SOLUTIONS.
IN ADDITION O THE COVID WORK WE
HAVE BEEN DOING.
WE TALKED TO SUMMARIZE IF YOU
WILL, WE TALK ABOUT HOW
TRANSITIONAL SCIENCE IS REALLY
PROVEN ITSELF OVER AND OVER TO
DRIVE INNOVATION AND LEAD
TRANSFORMATIONAL APPROACHES THAT
BRING US TO OUR ABILITY TO DO
MORE PREDICTIVE PRE-CLINICAL
PIPELINE DEVELOPMENT, TO EXPAND
OUR REPERTOIRE IN TECHNOLOGIES,
TO REVOLUTIONIZE DIAGNOSTIC
TREATMENTS TO PRIORITIZE DEI AND
ALL THE ACTIVITIES THAT WE
SUPPORT.
IN WAYS THAT WE TRAIN THE NEXT
GENERATION OF SCIENTISTS AND
ENGAGE OLD STAKEHOLDERS,
SHOULDN'T SAY OLD BUT PERHAPS
WHO HAVE BEEN OUR STAKEHOLDERS A
FILE AND FINDING NEW ONES WE
WANT TO BUILD THAT TRUST IN AND
NOSHTURE RELATIONSHIPS GOING
FORWARD.
THIS IS THE WAY TO BRING YOUR
TREATMENTS TO ALL PEOPLE MORE
QUICKLY.
THIS IS NOT JUST OUR MANTRA.
THIS IS OUR VISION.
WITH THAT VISION IT PROVIDED AN
OPPORTUNITY TO LOOK AHEAD O THE
NEXT DECADE, IN THE KEY NODE
ADDRESS DURING THAT EVENT I SET
FORTH THREE AUDACIOUS GOALS THAT
ARE FORWARD-LOOKING AND I WANT
TO SET A PATH NEXT DECADE HOW TO
OBTAIN THEM.
NCATS COUNCIL HEAR YOUR THOUGHTS
AND REACTS AND AS WE GET A
PERMANENT DIRECTOR, FOR NCATS
THERE WILL BE AN OPPORTUNITY TO
HONE AND DEVELOP AND METRACISE
THESE MORE.
BUT I WANT TO RUN LIEU THEM
BECAUSE ED I WOULD LIKE YOUR
REACTIONS SO GOAL NUMBER ONE,
MORE TREATMENT.
PERIOD.
RIGHT NOW WE HAVE 5% DISEASE
THAT HAVE A TREATMENT SHOWN HERE
IN THAT -- THIS BOTTOM BAR HERE
ON THE GRAPHIC WITH -- WE HAVE
ABOUT -- WE KNOW THERE'S OVER
7,000 RARE DISEASE TO DATE BUT
MORE ARE ON THE WAY.
WE ONLY HAVE 500 THERAPIES SO 5%
OF DISEASE HAVE A TREATMENT.
LET'S WORK TO GET THAT NUMBER UP
SO THAT A QUARTER OF KNOWN
DISEASE HAVE A TREATMENT IN THE
PIPELINE SO WE WENT 5% TO 25%.
WE ALSO KNOW 90% OF DRUGS IN
CLINICAL TRIALS PAIL BECAUSE OF
THE LACK OF EFFICACY AND SAFETY
CONCERNS.
IN VITRO ASSAYS THAT
RECAPITULATE HUMAN PHYSIOLOGY
AND PATHOLOGY SHOW INCREDIBLE
PROMISE BEING MORE PREDICTIVE OF
TOXICITY AND EFFICACY IN HUMANS
SO WE NEED TO REALLY ENABLE THAT
KIND OF DEVELOPMENT AND THOSE
TYPES OF TECHNOLOGIES TO HAVE A
WIDE ARRAY OF RESOURCES THAT
DON'T JUST MODEL TISSUES AND
ORGANS BUT MODEL DIVERSITY OF
INDIVIDUAL, MODEL GENETIC MAKE
UP, ENVIRONMENTAL EXPOSURES.
SO THERE'S MORE WORK TORR DO
HERE.
-- WORK TO DO HERE.
THERE'S OTHER INITIATIVES AS
WELL THAT WILL DEMOCRATIZE AND
DISSEMINATE NEW TREATMENT
APPROACHES, WE TALK ABOUT THE
PAGT PROGRAM AND THERAPY
CONSORTIUM.
THE SOMATIC CELL GENE EDITING
INITIATIVE; THESE ARE POISED TO
TRANSPERFORM THE PIPELINE FOR
GENE TARGETED THERAPIES BY
BREAKING DOWN THE BARRIERS OF
THE PRE-CLINICAL TO CLINICAL
APPROACHES BUT ALSO ADDRESS THE
MANUFACTURING AND THE REGULATORY
HURDLES IN THE PIPELINE AND SO
ANY RARE DISEASE CAUSE BY SINGLE
GENETIC MUTATION WE HOPE WILL BE
ABLE TO HAVE THE OPPORTUNITY TO
GET FIXED BY SOME OF THESE.
AND WHEN YOU THINK ABOUT 80% OF
THE RARE DISEASE ARE CAUSED BY
SINGLE GENE DISORDERS, THAT
MAKES IT SO THAT IF THIS ONLY
WORKS FOR HALF, THAT'S HUNDREDS
OF DISEASES.
THESE ARE WHY THE TYPES OF
PROGRAMS WHAT WE DO AT N CATS
WITH SRI AND ACADEMIA AND
PATIENTS, PATIENT ADVOCACY
GROUPS AND REGULATORS AND
FEDERAL PARTNERS WHO COME TO
IDENTIFY AND DIAGNOSE WHAT IS
CAUSING THIS SLOW PACE OF
TREATMENT AND DEVELOPMENT OF THE
TREATMENT AND WE ARE WORKING
HARD AND COLLABORATIVELY TO
BUILD PROGRAMS THAT WILL BRING
ABOUT THIS REVOLUTIONARY
TRANSFORMATION SO THIS IS A
MODEL THAT WE LOOK FORWARD TO.
GOAL TWO ALL PEOPLE.
WE WANT TO DRAMATICALLY INCREASE
INCLUSIVITY IN ALL AREAS, WORK
FORCE TRAINING RESEARCH CLINICAL
TRIALS AND HEALTH OUTCOMES.
AND JUST WHEN YOU THINK ABOUT
COVID, VERY RECENT AND STARK
EXAMPLES, ARE COMING OUT FROM
COVID FOR SURE, BUT THE COVID-19
OCCURRENCE OF DEATH AMONG RACIAL
ETHNIC POPULATIONS IN RURAL
COMMUNITIES IN OUR COUNTRY HAVE
BEEN ONE OF THE MOST PAINFUL
ACTS TO SEE DURING THIS
PANDEMIC.
BLACK AND AFRICAN AMERICANS
HISPANIC AND LATINO, AMERICAN
INDIAN AND ALASKA NATIVE ARE
CONTRACTING AND DYING FROM
COVID-19 THAN WHITE ASIAN
COUNTER PARTS IN THOSE
POPULATIONS.
THERE WAS A RECENT PRE-PRINT OUT
OF THE NIGMS CTR PROGRAM USING M
3C SHOWING RATES IN RURAL
COMMUNITIES ARE 40% HIGHER THAN
IN OTHER COMMUNITIES.
THIS IS A BIG DEAL.
WE NEED TO ADDRESS IT, WE NEED
TO DO BETTER.
CTSA ACROSS THE COUNTRY
PRIORITIZED ENGAGEMENT
PARTNERSHIPS AND INNOVATING
AROUND STRATEGIES THAT MEET
PEOPLE WHERE THEY ARE, MOBILE
VANS, COMMUNITY, CENTERS PLACES
OF WORSHIP, GROCERY STORES
LIBRARIES, ALL THOSE SORTS OF
THINGS.
THESE EFFORTS ARE MADE CTSA
CORNERSTONE OF THE TRUSTED
COMMUNITY PARTNERS THAT HAVE AND
WE NEED TO ENABLE THEM MORE TO
PIVOT RAPIDLY ON A VARIETY OF
HEALTH OUTCOMES WE ARE
INTERESTED IN PURSUING AS WELL.
TALK ABOUT DEIA EFFORTS A BIT
AGO AND THE POINT IS THAT
STUDIES OF TOMORROW, WHAT WE DO
IN THE NEXT DECADE ENSURE NO ONE
IS LEFT BEHIND IN OUR PLANNING
DESIGNING OUR IMPLEMENTING.
RESEARCH IMPROVES LIVES FOR ALL.
INCREASING DIVERSITY IN ALL WAYS
IS GOAL NUMBER TWO.
GOAL 3, MORE QUICKLY, WE WANT TO
REDUCE THE TIME IT TAKES FOR A
TREATMENT TO REACH THE MARKET BY
HALF.
IT TAKES 10 TO 15 YEARS AND
$2.6 BILLION FOR DRUG TO MAKE IT
TO MARKET.
WHICH NEED REDUCE THAT TIME.
TO DO THAT WE NEED TO FAIL FAST,
EARLIER AND IMPORTANTLY WE NEED
TO FAIL FORWARD AND THAT IS A
KEY FACTOR BECAUSE WHEN YOU FAIL
FORWARD YOU ARE TRIPPING OVER
THE VULNERABILITIES THAT WE HAVE
FOUND AND YOU CAN FIX THEM BUT
YOU -- WHEN YOU FAIL FORWARD,
YOU NOT HOLDING YOURSELF BACK
YOU ARE FAILING FORWARD TO MAKE
SURE YOU IDENTIFY THOSE
VULNERABILITIES AND MITIGATE
THAT RISK.
IT IS IMPORTANT TO DO THAT.
WE WANT TO PROMOTE EMPLOY MORE
STRATEGIES ADDRESSING MANY
DISEASES AT A TIME AND FINDING
WHAT IS COMMON ACROSS DISEASE
AND TARGETING THOSE UNDERLYING
MECHANISMS.
ON TOP OF THAT HAVE TRAINING THE
NEXT GENERATION OF SCIENTISTS IS
HUGELY IMPORTANT TO HELP RETHINK
HOW WE TRAIN OUR SCIENTISTS AND
ENCOURAGING TO THINK MORE
REVOLUTIONARY AND
TRANSFORMATIONAL TERMS AS
OPPOSED TO MORE EVOLUTIONARY
TERMS.
FINDING COLLABORATION AND PART
SHIPS ARE CRITICAL FOR NCATS AND
WHAT WE DO AND WHEN WE THINK
ABOUT EXECUTING AGREEMENTS THOSE
CAN TAKE TIME SO WHAT WE HAVE
BEEN ABLE TO DO IS QUICKLY
LAUNCH COLLABORATION BECAUSE WE
USE COLLABORATIVE THEM PLACE OR
AGREEMENT THEM PLACE THAT ALLOW
COLLABORATIONS TO HAPPEN ON
ORDER OF HOURS, DAYS SOMETIMES
BUT THERE'S BEEN A COUPLE OF
THEM DONE ON HOURS AND NOT
MONTHS.
SO THIS TIME SAVING IS HUGELY
IMPORTANT FOR US.
THE COVID SCENARIO FOR THE
CLINICAL TRIALS HAVE REALLY
IDENTIFIED HOW MASTERED UMBRELLA
PROTOCOLS ARE REALLY HELPING US
TO BE CLINICAL TRIAL READY.
THINKING SINGLE IRB REVIEW
BOARDS AND ALLOWING THEM TO
ACTIVATE MORE EFFICIENTLY AND
ENROLL PARTICIPANTS SOONER,
THESE ARE THE KEY ACTIVITIES.
AT THE LAST COUNCIL I TALK DATA
SCIENCE EFFORTS AND NEW TOOLS TO
SEE PATTERNS OF BIG DATA THAT WE
HAVEN'T SEEN BEFORE.
OF COURSE N 3C SHOWS US WE CAN
CATALYZE THE HEALTH DATA AS A
PRINCIPLE THAT IS SOMETHING TO
PUSH FOR ALL DISEASES.
THINKING OTHER APPROACHES LIKE
DRUG REPURPOSING, OF COURSE IF
YOU CAN DO DRUG REPURPOSING CUT
THAT TIME LINE TO COUPLE OF
YEARS AS OPPOSED TO TEN YEARS
THAT IT TAKES TO DEVELOP DRUGS
BECAUSE WE KNOW A LOT ABOUT
THOSE DRUGS BEING REPURPOSED,
BEEN FDA APPROVED FOR EXAMPLE SO
OTHER WAYS TO GAIN E FORB SHIES
SO A LOT OF -- EFFICIENCIES.
SO THESE ACTIVITIES ARE AROUND
THAT APPROACH AND GOALS ARE
ASPIRATIONAL BUT THE WHOLE IDEA
IS TO BRING MORE TREATMENT TO
ALL PEOPLE.
AS I TALK TO OUR INTERNAL TEAM
MORE ON THESE IDEAS IT IS CLEAR
TO ME WE CAN MAKE HEADWAY ON
THEM.
IMPORTANTLY THE GOALS WE
FINALIZE WILL GUIDE AND -- US TO
THE NEXT DECADE AND KEEP US
FOCUSED ON MAKING THESE GOALS A
REALITY.
SO I PLAN TO USE THESE TO ALSO
HELP FRAME SOME OF THE SUCCESS
THAT WE HAVE MADE, AND WITH THAT
I'M INTERESTED IN YOUR THOUGHTS
ON ALL THINGS WE DO TODAY AN
ESPECIALLY AROUND THESE THREE
AMBITIOUS AND AUDACIOUS GOALS.
I WILL STOP HERE AND I'M GOING
TO TURN IT OVER TO CLAIRE AND
PENNY TO MANAGE THE DISCUSSION.
THANK YOU VERY MUCH.
I APPRECIATE YOUR ATTENTION.
>> GREAT, JONI.
THANKS SO MUCH.
MS. KENNEDY, WOULD YOU LIKE TO
VERBALIZE THE COMMENT YOU PUT IN
THE CHAT BOX?
>> SURE, I ALSO HAVE A QUESTION
IF I CAN JUMP IN WITH THAT.
I WANT TO THANK THOSE WHO ARE
STEP DOWN FROM THEIR ROLES AND
NCATS AND ESPECIALLY DR. PARISER
ONER HER RETIREMENT FROM PUBLIC
SERVICE AT NCATS AND PREVIOUSLY
AT FDA WITHOUT A DOUBT HER
SERVICE CHANGED OUR RARE DISEASE
LANDSCAPE FOREVER SO I THINK WE
NEED TO RECOGNIZE THAT AND
ACKNOWLEDGE THAT WE HAVE A DEPTH
OF GRATITUDE TO YOU.
WE ARE REALLY EAGER TO CONTINUE
TO WORK WITH DR. BROOKS WE KNOW
A LOT OF MOMENTUM IN ORDR IS
BECAUSE OF SHARED LEADERSHIP SO
LOOKING FORWARD TO WORKING WITH
BOTH OF YOU THROUGHOUT THIS
TRANSITION AND DR. BROOKS IN THE
MONTHS AND HOPEFULLY YEARS
AHEAD.
THANK YOU.
I DID HAVE A QUESTION, IF I CAN
DO THAT AS WELL.
SO FIRST, THANK YOU FOR THIS
WONDERFUL PRESENTATION, I FEEL
LIKE I GET TO MAYBE HEAR YOU
MONTHLY AND EVERY TIME THERE'S A
LOT OF NEW INFORMATION THAT YOU
BRING FORWARD AND A LOT OF GREAT
PROGRESS.
A COUPLE OF THINGS YOU PRESENTED
SPEAKS OUT TO ME AS AGILITY OF
NCATS AND THE ABILITY TO NCATS
TO PIVOT.
ONE EXAMPLE YOU GAVE WAS IN A
CONVERSATION WITH DR. WOODCOCK,
A CONCEPT AND IDEA SHE HAD HAS
NOW COME TO FRUITION AND YOU ARE
SOON GOING TO HAVE DATA AROUND
REAL WORLD EVIDENCE.
UTILIZING MONOCLONAL ANTIBODIES.
THAT IS A QUICK PIVOT.
ONE THOUGHT WE DON'T HAVE TO
ANSWER HERE BUT ONE QUESTION IS
HOW DO WE BETTER COMMUNICATE NOT
JURY OUR IMMEDIATE STAKE HOLDERS
BUT THE GENERAL PUCKLY THE
IMPACT NCATS HAS ON ANSWERING
SOME OF OUR URGENT PUBLIC HEALTH
ISSUES.
AND ADDRESSING SOME OF THOSE.
SO THAT SERVES SORT OF STRIKESES
ME AS NCATS IS ONE OF OUR BEST
KEPT SECRETS AND ONE OF THE MOST
POWERFUL RESOURCES AND TOOLS.
AS YOU WERE PRESENTING THAT
JUMPED OUT AT ME TIME AND AGAIN.
THANK YOU FOR THE WORK YOU ARE
LEADING.
>> THANK YOU SO MUCH, ANNIE.
ECHOING YOUR THANKS TO ANN
PARISER, WHICH I COULDN'T AGREE
MORE, YOUR QUESTIONS, I DON'T
KNOW HOW TO ANSWER YOUR
QUESTION.
EXCEPT TO SAY THAT AGREE WE NEED
TO THINK ABOUT WAYS TO
COMMUNICATE THIS KIND OF
INFORMATION, I THINK AT THIS
STAGE HOW WE ARE GATHERING THIS
INFORMATION AGAIN THE ONE I
PRESENTED IN THERE WILL BE
OPPORTUNITIES FOR US TO DO
CERTAINLY PRESS RELEASES TO GET
THAT INFORMATION OUT MORE AND
BUT I THINK THERE IS MORE TO
LEARN TOO IN TERMS OF HOW WE
THINK ABOUT WHO IS GOING TO BE
AT RISK OR HOSPITALIZATION, WHO
WE NEED TO TARGET FOR THESE
KINDS OF THERAPIES.
AND NOT ONLY THAT, YOU MENTIONED
THE PUBLIC COMMUNICATIONS BUT
THERE'S ALSO THE PHYSICIAN
COMMUNICATION, MONOCLONAL
ANTIBODIES ARE NOT REALLY A
WIDESPREAD THERAPEUTIC IN MANY
SPACES SO THERE'S PHYSICIANS WHO
DON'T KNOW A LOT ABOUT
MONOCLONAL ANTIBODIES AND HOW
THEY ARE USED AND WHY USEFUL FOR
COVID-19.
SO A LOT OF THE WORK THAT I
THINK WE ARE FINDING IS HOW DO
WE DO WE COMMUNICATE TO
STAKEHOLDERS AND PHYSICIAN
COMMUNITY SO THEY UNDERSTAND HOW
TO USE THESE TYPES OF THERAPIES
AND WHEN TO USE THEM AS WELL,
RECOGNIZING THAT WITH DIFFERENT
VIRAL VARIANTS YOU MIGHT HAVE
DIFFERENT RESPOND -- HAVE
DIFFERENT RESPONSES TO THE
MONOCLONAL ANTIBODIES.
SO THAT IS ONE THING.
THE OTHER IDEA WE HAVE, I THINK
WE DO A REALLY GOOD JOB
COMMUNICATING TO SCIENTIFIC
COMMUNITY, THROUGH THE OPEN DATA
PORTAL BECAUSE PEOPLE CAN
UNDERSTAND HOW TO READ THAT
INFORMATION BUT WE TRY TO DO A
GOOD JOB IN HOW WE DISPLAY THAT
DATA TO MAKE IT AS EASILY
DIGESTIBLE AS POSSIBLE.
THE OPEN DATA PORTAL IS A
WONDERFUL EXAMPLE OF THAT, IT IS
EASY TO COMMUNICATE THAT.
THEN THE QUESTION IS HOW DO WE
THINK ABOUT COMMUNITIES AND I
WOULD LOVE TO HEAR IDEAS THIS
GROUP MIGHT HAVE TO BE ABLE TO
DO MORE OUTREACH ON EDUCATING
FOLKS AND PERHAPS THAT IS MORE
WEB PAGES BUT THERE ARE OTHER
MORE ACTIVE KINDS OF THINGS THAT
WE CAN DO.
SO I LIKE THAT IDEA, AS WE
TALKED ABOUT MORE RECENT DATA
FROM MONOCLONAL ANTIBODY DATA,
WE ARE SEEING THAT SAME IDEA OF
HOW DO WE THINK ABOUT THIS
INFORMATION OUT THERE,
DISSEMINATION IS A KEY ASPECTS
OF WHAT WE DO.
SO WE ARE TRYING TO FIGURE OUT
THAT AS WELL.
THAT IS A VERY GOOD POINT.
>> DR. CRESLER WOULD YOU LIKE
THE GO?
>> THANK YOU FOR SHOWING HOW
NCATS IS RESPONDING TO THE ACUTE
NEEDS AT HAND AND STILL KEEP
LONG TERM STRATEGY IN HAND AND
REALLY WANT TO 5 TO 25% PUSH FOR
DEVELOPING TREATMENTS.
IT SOUNDS AUDACIOUS TO ACTUALLY
IMPOSSIBLE TO DO AND I WILL
COUNT ON THAT.
BECAUSE WHAT WE ARE SEEING RIGHT
NOW IS THAT WE ARE DEFINING AND
REDEFINING HOW WE WORK WITH
HUMANS AS OUR HEAT MAP AND CELL
BASED -- WE ARE ALIGNING CIVIL
AND MACHINERY AND MOST
IMPORTANTLY WHY THEY FAIL.
WHY IT MIGHT NOT BE POSSIBLE TO
DEVELOP SPECIFIC TARGETED
THERAPIES FOR TWO AND A HALF
THOUSAND DISEASES AND I THINK WE
HAVE ALL THE INSTRUMENT TIME AT
HAND AND NCATS HAS THE TOOLS TO
DEPLOY THEM, TO IDENTIFY SHARE
COMMON PATHWAYS OF DISEASE
MANIFESTATIONS WHICH CAN PREVENT
A LESION WHICH WOULD CAUSE A
PHENOTYPE IN THE PATIENTS TO
REMAIN SUB CLINICAL.
I KNOW THAT THAT HAS BEEN
DISCUSSED INSIDE NCATS FOR QUITE
A WHILE IN SEVERAL KNOWLEDGE
ACTIVITIES, I HEADED THAT WAY
BUT IT IS RARELY -- REALLY
IMPRESSIVE TO SEE HOW QUICKLY
DISEASE SPECIFIC ACTIVITIES IN
THE CELL MAPPING WE ARE MAKING
FOCUS TO IDENTIFY THESE PATIENT
SPECIFIC CHANNELS AND HOW TO
ENTERPEER WITH THEM IN A TRANS--
INTERFERE WITH A TRANSLATIONAL
SENSE.
THIS IS THE INSTITUTE WHO
LEVERAGE ALL THEIR KNOWLEDGE AND
EXOSOME DISCUSSIO DISCUSSIONS WITH VERY
SPECIFIC APPLICATION ALREADY BUT
I THINK WHATEVER YOU CAN PUSH
BACK, THIS IS JUST ARBITRARY
NUMBER, NO IT ACTUALLY ISN'T.
>>
>> SORRY ABOUT THAT MA,THIAS.
THANK YOU SO MUCH FOR THOSE
COMMENTS.
I WAS WORRIED TOO THAT IT MIGHT
BE -- THAT 25% NUMBER MIGHT BE
TOO BIG BUT AT THE SAME TIME I
AGREE WITH YOU.
I THINK WE HAVE TO GO FOR IT.
YOUR POINT ABOUT THE SUB
CLINICAL UNDERSTANDING OF THOSE
END POINTS IS ALSO IMPORTANT AND
I THINK WITH THE IN VITRO ASSAYS
THAT WE CAN PUT TOGETHER
PRINTING AND TISSUE CHIPS WE ARE
USING, IT IS ALSO TO START
IDENTIFYING WHAT THE SUB
CLINICAL PHENOTYPES ARE AS
BIOMARKERS THAT CAN BECOME
SOMETHING USED CHUNKILY AND AN
AREA WE WILL TRY TO DRIVE
FORWARD AS WELL.
APPRECIATE YOUR COMMENTS.
>> THAT WAS TERRIFIC.
THANK YOU SO MUCH.
WHAT I WOULD LIKE TO EMPHASIZE,
WE ARE ALMOST SEEING EMERGENCE
OF NEW TYPE OF FIELD OF
MEDICINE.
WHICH IS AROUND SMALL PATIENT
GROUP POPULATIONS WHO HAVE YOU
BEEN SUBDIVIDED BY MOLECULAR AND
OTHER TYPES AND THE RARE DISEASE
FIELD THAT I LIVE IN, WE ARE
LITERALLY SEEING 11 NEW
CONDITIONS A WEEK.
WHEN YOU LOOK AT THE LINKING OF
GENETIC VARIATION TO THAT.
THAT REALLY DOESN'T FIT THE
CLASSIC MODEL, THE STATISTICS
ARE DIFFERENT, THE EVIDENTIARY
PROOF IS DIFFERENT.
THE CLINICAL TRIALS MODELS AND
THERE'S REALLY M CATS IS BETTER
POSITIONED TO DEAL WITH THIS AND
TO LEAD THE WAY THAN ANY OTHER
GROUP.
BECAUSE IT IS DISEASE AGNOSTIC
TO A CERTAIN EXTENT.
THE METHODOLOGIES THAT APPLY TO
THESE SMALL GROUPS CAN GO ACROSS
MULTIPLES BUT WE NEED A HOMING
TOOL -- WE ARE BUILDING A
TOOLBOX, WE HAVE TO CONTINUE TO
BUILD THAT.
SO THE ONLY THING I WOULD ADD ON
TOP OF THAT VERY GOOD
PRESENTATION IS BUCKLE UP BUT
ALSO IT IS VERY IMPORTANT TO
ENGAGE THE FDA CONSISTENTLY AND
ACTIVELY.
BECAUSE IF WE DON'T A LOT OF THE
WORK THAT WE DO IS GOING TO BE
WAISTED WHEN PEOPLE HAVE TO GO
BACK AND USE A DIFFERENT CLASSIC
MODEL WHEN TAKING TRIALS THROUGH
FOR APPROVAL.
>> THAT IS A GREAT POINT,
MARSHALL.
THANK YOU FOR BRINGING UP THAT
SPECIFIC ISSUE AROUND THE FDA
AND I'M GRATEFUL FOR THE WORK
THAT THE FDA DOES AND THEY --
THEY WORK HAND IN HAND WITH US
ALONG THE WAY.
CASE IN POINT THAT I WOULD SAY
IS WITH THE SPOKE GENE THERAPY
CONSORTIUM, THIS IS ANOTHER ONE
OF THESE PUBLIC PRIVATE
PARTNERSHIPS THAT WE DO WITHIN
THE NIH DUE TO THE ACCELERATED
MEDICINE PROGRAM.
FOR THAT PARTICULAR PROGRAM FDA
IS IN IT FROM THE BEGINNING.
AND THEY ARE HELPING US BUILD IT
AND DEVELOP THE FRAMEWORK FOR IT
BECAUSE IT IS NOT JUST ABOUT THE
SCIENCE THAT WE NEED TO SUPPORT
IN TERMS OF UNDERSTANDING THE
ADENOASSOCIATED VIRAL THERAPIES
BUT IT IS ALSO THAT WE NEED TO
FIGURE OUT THAT MODEL OF HOW TO
WORK THROUGH REGULATORY SPACE SO
THAT IT IS NOT JUST A ONE OFF,
IT IS A PLATFORM TECHNOLOGY.
I THINK THE DISCUSSION WE HAVE
HAD WITH PETER MARKS AT THE FDA
HAVE BEEN REALLY EXCITING THAT
THEY ARE VERY MUCH INTO THAT
KIND OF IDEA AND WE LOOK FORWARD
TO WORKING THROUGH THAT THAT
PLATFORM IN AND OF ITSELF FOR
SPOKE GENE THERAPY PROGRAM BUT
IF IT WORKS IT BECOMES A BIGGER
PLATFORM WHICH YOU CAN APPLY
OLGONUCLEOTIDE TYPES OR OTHER
THINGS SO IT WILL BECOME A
PLATFORM OF PLATFORMS AND I
THINK THAT'S REALLY EXCITING.
>> ONE MORE THING TOO, I SAT ON
COUPLE OF FDA COMMITTEES WHERE
WE TALK ABOUT HOW WE IN THE
MODERN AGE HOW WE DO CLINICAL
TRIALS AROUND THESE.
THERE'S ANOTHER SIDE TOO, NOT
JUST WHAT WE ARE GIVING TO
PATIENTS IS HOW WE ARE TESTING
THE OUTCOMES.
WE NEED A NEW SET OF TOOLS THAT
ARE MORE DESIGNED AROUND
PATIENTS WHO ARE GEOGRAPHICALLY
SEPARATED IN OTHER WORDS ONLINE
DIGITAL TOOLS AND COVID TAUGHT
US NOTHING ELSE CONCERNING MOST
EVERY NIH PROTOCOL WAS FULL OF
PROTOCOL VIOLATIONS BECAUSE
PATIENTS COULDN'T COME IN ANY
MORE FOR THINGS BUT NONE OF THE
TOOLS ARE VALIDATED SO THERE IS
A LOT OF ROOM FOR PARTNERSHIP ON
DEVELOPING TOOLS, VALIDATING THE
TOOLS.
SO THAT NOT ONLY CAN WE PROVIDE
PLATFORMS LIKE OUR GENE THERAPY
BUT ALSO WE HAVE WAYS TO SAY
OKAY IS IT EFFICACIOUS, AND CAN
WE DO THAT IN A WAY THAT ISN'T
GOING TO CRUSH THE FAMILIES AND
PATIENTS.
THEY ALREADY GOT ENOUGH ON THEIR
PLATE ALREADY.
>> ABSOLUTELY.
AND AS YOU KNOW, THEY ARE THE
ONES DOING ALL THE MONEY RAISING
AND FOR HERMING THOSE PARTICULAR
DISEASE, IF THIS WORKS IT WOULD
BE A HUGE BURDEN OFF OF THEIR
SHOULDERS.
>> VERY EXCITING.
THANK YOU.
>> DR. HARRIS, YOU ARE NEXT.
>> THANK YOU.
ECHO OTHER COMMENTS THAT GREAT
PRESENTATION, LOVE THE GOALS.
I SORT OF FOCUS ON THE MORE
PEOPLE AND THINKING ABOUT THE
FACT THAT GIVEN WORK YOU GUYS
HAVE DONE WITH N 3C NHLBI HAS
DONE WITH BIODATA CATALYST AND
THIS IS A NEW ERA WE ARE IN AND
IT DEMOCK TIDESES ACCESS TO SOME
RESOURCES THAT WERE UNHEARD OF A
DECADE AGO.
BUT I THINK TO MAKE IT REALLY
WORK, WE NEED TO THINK ABOUT
INVESTING IN THE WORK FORCE AND
MAKING SURE THAT PEOPLE ARE DATA
PROFICIENT AND INVEST IN WHAT
YOU CALL TRANSLATORS OR MATCH
MAKERS, BEING ABLE TO PAIR DATA
SCIENTISTS WITH CLINICAL EXPERTS
WITH COMMUNITY ENGAGEMENT
EXPERTS, ET CETERA.
THAT IS GOING TO BE NEEDED TO
TAKE US FROM WHERE WE ARE TO
WHERE WE COULD BE BUT SO JUST
ENCOURAGE US ALL TO BE THINKING
STRATEGICALLY ABOUT THAT SORT OF
WORK.
>> GREAT IDEA.
WITHIN THE N 3C WHAT I DON'T
TALK ABOUT TOO MUCH IS THERE ARE
THINGS CALLED DOMAIN TEAMS.
THE IDEA BEHIND IT, IT'S GETTING
TO THAT, IT IS ABOUT PAIRING
STATISTICIAN WITH CLINICIAN ON A
PARTICULAR OUTCOME THEY MIGHT BE
LOOKING AT BUT A STATISTICIAN IS
NOT GOING TO KNOW WHETHER IS
IMPORTANT IN TERMS OF
UNDERSTANDING END POINTS SO
DOING THAT KIND OF PAIRING IS
GOOD BUT WHAT YOU ARE SAYING IS
THAT -- THAT MIGHT BE NECESSARY
BUT IT IS NOT SUFFICIENT.
I THINK YOU ARE RIGHT, WE HAVE
BEEN THINKING ABOUT THIS, WE
HAVE BEEN PARTNERING WITH NIH
AND THE OFFICE OF DATA SCIENCE
AND STRATEGY, THEY ARE DOING A
VARIETY OF PROGRAMS ON SOMETHING
CALLED IN THE HEAD TRYING TO
MAKE SURE THEY ARE TOOLS AND
RESOURCES AVAILABLE FOR ALL
COMMUNITIES TO BE MORE DATA
PROFICIENT BUT EVEN THERE I FEAR
IT IS NECESSARY BUT NOT
SUFFICIENT SO THAT IS SOMETHING
TO KEEP UPDATED ON, IT IS A VERY
ASTUTE POINT AND I WROTE IT
DOWN.
THANK YOU.
>> DR. JACKSON.
>> HI, JONI.
THANK YOU FOR THAT OVERVIEW AND
TOUCHING SOME OF THE HIGHLIGHTS
OVER THE LAST SEVERAL MONTHS.
I GUESS I WANT TO FOLLOW-UP ON
PAUL.
SO I HAVE TO MAKE SURE I RAISE
MY HAND BEFORE PAUL SO HE
DOESN'T FEEL WHAT I WAS GOING SO
SAY BUT IT IS ALSO AROUND SAME
CONCEPT OF ALL PEOPLE AND DATA
DEMOCRATIZATION.
I WONDER WHAT ARE KIND OF THE
EFFORTS THAT ARE GOING TO BE
GOING FORWARD WITH N CATS
SPECIFICALLY AROUND DATA EQUITY
ACCESSIBILITY PARTICULARLY FOR
COMMUNITY STAKEHOLDERS, AND
OTHERS REDUCING BIAS WITH DATA
FROM INCEPTION TO FINAL
ANALYSIS.
THEN ADDRESSING SOME OF THE
ISSUES AROUND DIGITAL EQUITY AS
WELL.
ARE THESE AREAS THAT NCATS IN
FACT TAKE SOME LEADERSHIP BOTH
AROUND THE RARE DISEASE BUT
BROADLY AROUND TRANSLATIONAL
SCIENCE, BECAUSE IT IS THE
QUALITY OF OUR DATA, DON'T
INCLUDE KIND OF THE BREADTH OF
OTHER THINGS THAT IMPACT HUMAN
HEALTH, THEN WE WILL CONTINUE TO
MAKE ERRORS AS WE MOVE FORWARD
NOT UNDERSTANDING EVERYTHING
FROM EXOSOME ALL THE WAY THROUGH
SOCIAL DETERMINANTS OF HEALTH OR
EVEN ISSUES ASSOCIATED WITH
STRUCTURAL RACISM.
>> THANK YOU FOR BRINGING THAT
UP.
THE SHORT ANSWER IS THAT AT
NCATS WE HAVE JUST STARTED TO --
MAYBE THIS IS THE LONG ANSWER.
SHORT ANSWER YES.
THE LONG ANSWER THOUGH IS MORE
INTERESTING.
WE JUST STARTED TO BUILD A
REALLY DEEP AND WIDE FOCUS ON
DATA SCIENCE.
AND WE HAVE A DATA SCIENCE LEAD
THAT IS MANAGING ACROSS THE
NCATS AND A LITTLE BIT ABOUT
WHAT WE HAVE COME UP WITH, WE
HAD A RETREAT TO TALK ABOUT DATA
SCIENCE AND WE ARE NOW HAVING A
FOCUSED EFFORT IN THE TYPES OF
DATA SETS AND RESOURCES THAT WE
HAVE.
ABLE TO MAKE SURE THAT --
AVAILABLE.
TO MAKE SURE WE ARE TALKING
ABOUT THE IDEA OF TRANSPARENCY
AND WHAT IS IN THE DATA HOW TO
UNDERSTAND WHAT IS IN THE DATA.
ALSO TO MAKE SURE THAT WE CAN
IDENTIFY BIASES IF THEY ARE IN
THE DATA.
IF YOU KNOW AND IDENTIFY YOU CAN
INTERPRET THINGS BETTER.
THE WHOLE IDEA OF COURSE THEN
TOO IS UNDERSTAND WHAT BIASES
ARE SO YOU CAN ADDRESS THEM AND
BRING MORE DATA INTO EVALUATE
THAT.
I WILL SAY ONE OF THE THINGS FOR
THE N 3C WE HAVE BEEN WORKING
ON, IS A MORE CONCRETE EXAMPLE
WE REALLY WANTED TO WORK WITH --
BECAUSE THE DATA TYPES THEY ARE
BRINGING IN ARE AREAS MORE
UNDERSERVED SO WE WANTED TO MAKE
SURE WE -- THAT TYPE OF DATA WAS
REPRESENTED IN THE N 3C COHORT.
EVEN THEN I THINK WE STILL NEED
TO DO A BETTER JOB AND ENSURE
THAT WE ARE GETTING THAT
DISTRIBUTION AND REPRESENTATION
OF THE UNITED STATES SO THAT WE
HAVE THAT DATA TRANSPARENCY SO
THAT THINGS WE INTERPRET FROM
THE STUDIES THAT WE DO ARE
GENERALIZABLE IN A WAY.
AND I THINK THAT IS SO
IMPORTANT.
AND THIS GOES BACK TO WHY I
THINK I'M EXCITED ABOUT THE
TRIBAL CONSULTATION WE ARE ABOUT
TO HAVE THAWS BAY IS ANOTHER
AREA WE NEED TO BOLSTER AND
SUPPORT BUT ALSO RECOGNIZE THAT
THE -- THEY HAVE TRIBAL
SOVEREIGNTY OVER DATA THEY HAVE.
AND WE WANT TO MAKE SURE WE ARE
HONORING THAT AND RECOGNIZING
THAT.
SO WE WANT TO USE THAT VERY
WELL, AND SO THAT THAT COMMUNITY
THEN CAN TAKE A LOOK AT THE DATA
AND APPLY IT NECESSARILY TO HELP
THEM THINK ABOUT WHAT KINDS OF
RESOURCES THEY MIGHT NEED GIVEN
SPECIFIC SCENARIOS IN THAT
COMMUNITY AND THINK ABOUT DOING
THAT AS WELL.
SO THIS IDEA OF BIAS IS
SOMETHING WE THINK ABOUT AND
TALK ABOUT A LOT AND I AM --
THIS IS -- I THINK OVER THE NEXT
YEAR AT N CATS DATA SCIENCE AND
THIS PARTICULAR IDEA IS ONE OF
THE BIGGER THINGS THAT WE WILL
BE FOCUSING ON.
>> MS. HARTMAN YOU ARE NEXT.
>> THANK YOU.
>> FEW FOR THE FAIR WELL, I'M
SAD THIS IS MY LAST MEETING BUT
LOOK FORWARD TO WORKING WITH ALL
OF YOU MANY LEADING GOVERNMENT
AFFAIRS FOR ALLIANCE FOR
REGENERATIVE MEDICINE SO STILL
IN THIS SPACE BUT I WANT TO
COMMENT MORE WEARING MY HAT AS A
RARE MOM, ONE OF THE MOST
IMPORTANT THINGS I HAVE SEEN
WORKING WITH NCATS HAS BEEN
CHAMPIONSHIP RARE DISEASE.
ANNIE SAID QUITE A BIT ABOUT
THIS AND ECHOED COMMENTS AND
SAME WITH MARSHALL SUMMERS RARE
IS A PUBLIC HEALTH PRIORITY.
THIS HAS BEEN AN ISSUE THAT WE
HAVE BEEN TRYING TO PUSH WITH
THE ASSISTANCE FUND WORKING
CLOSELY WITH DR. SUMMER AND
CHILDREN'S NATIONAL WORKING
CLOSELY WITH ANNIE KENNEDY AND
HER COLLEAGUES AT THE EVERY LIFE
FOUNDATION AND THE REST OF THE
RARE DISEASE COMMUNITY.
NCATS HAS ALWAYS BEEN THE
BIGGEST CHAMPION FOR RARE
DISEASE.
JONI YOUR LEADERSHIP IN THIS
SPACE IS CRITICAL AND WE HOPE
THIS WILL CONTINUE.
PERSONALLY AND PROFESSIONALLY
VERY IMPORTANT TO ME THAT THIS
CONTINUES.
I KNOW WE WILL HEAR FROM TARA ON
ARPA H AND THIS IS AN ISSUE THE
RARE COMMUNITY IS FOCUSED ON HOW
TO ENSURE THAT RARE DISEASES ARE
ALSO PRIORITIZED WITHIN ARPA H
THE RARE DISEASE COMMUNITY THE
ASSISTANCE FUND EVERY LIFE
GLOBAL GENES RARE -- THE WHOLE
SLEW OF ORGANIZATIONS PUT A
WHITE PAPER TOGETHER, HAPPY TO
SHARE IF YOU HAVEN'T SEEN IT, IT
IS VERY SHORT BUT OUTLINES WHAT
RARE DISEASE COMMUNITY WOULD
LIKE TO SEE, ARPA H FOCUS ON AND
WORK WITH NCATS GOING FORWARD.
SOME OF THE RARE PARENTS AND I
HAVE HAD A THE CUSHION ABOUT
WHAT WAS RAISED IN -- DISCUSSION
ABOUT WHAT WAS RAISED IN TERMS
OF NIH AND FDA WORK CLOSE
TOGETHER AND MAKING SURE THEY
ARE WORKING WITH CMS SO THAT
ONCE THESE DRUGS HIT THE MARKET
PATIENTS CAN ACCESS THEM AS WELL
AND THAT IS SOMETHING WE SPENT
TIME ON AT THE ASSISTANCE FUND.
WHEN IT COMES TO EVEN GETTING
THESE DRUGS INTO THE PIPELINE
AND THROUGH THE FDA, ONE
CONVERSATION WE HAVE BEEN HAVING
AS CAREGIVERS OF RARE DISEASE
QUITE A BIT IS THE NEED FOR A
NEW LOOK AT THE RISK CALCULATION
THAT FDA IS USING.
THIS IS SOMETHING A LOT OF US
HAVE SPOKEN PUBLICLY ABOUT BUT
THERE NEEDS TO BE A LARGER
CONVERSATION.
WHEN CHILDREN ARE DYING, THAT IS
SOMETHING THAT THE FDA DOES
UNDERSTAND.
MANY OF YOU MAY REALIZE WITH MY
SITUATION MY DAUGHTER HAS A
SEVERE DEVELOPMENTAL DISABILITY.
SO SHE'S ESSENTIALLY A PERPETUAL
TODDLER, SHE CAN BARELY WALK,
SHE CAN'T TALK, SHE'S GOING TO
BE SIX SOON, SHE CANNOT COMPLETE
ANY OF THE ACTIVITIES OF DAILY
LIVING WITHOUT ASSISTANCE.
IT IS VERY MUCH LIFE ALTERING.
THE RISK CALCULATION FOR US IS
ALSO VERY DIFFERENT.
WE -- THERE WAS JUST A
CONVERSATION THAT LUKE ROSEN
RELEASED ABOUT HOW CAREGIVERS
WOULD LIKE TO TIME OUR LEAVING
THIS PLANET WITH OUR CHILDREN
BECAUSE THE THOUGHT OF LEAVING
THEM HERE ALONE IS TERRIFYING
BECAUSE WE ARE NOT SET UP IN
THIS SOCIETY TO CARE FOR THEM.
IT IS IMPORTANT TO CONSIDER THIS
RISK CALCULATION.
FOR THESE CHILDREN AS WELL.
BECAUSE RIGHT NOW, WE DON'T HAVE
A GOOD SOLUTION.
A LOT OF US ARE AWAKE A LOT OF
NIGHTS THINKING ABOUT THIS.
THE HOPE THAT THE SCIENCE WILL
MOVE FORWARD IS CRITICAL.
I'M ALSO HOPEFUL THAT WE'LL DO
MORE WORK AND THIS IS NOT NCATS
JOB BUT LOOKING HOW WE DO TAKE
CARE OF THESE CHILDREN.
THERE IS NOT ENOUGH OF THE -- IN
THE WORLD TO CARE FOR THEM.
WE DON'T WANT TO HAVE ANOTHER
WILLOW BUTCH, THE INSTITUTION
WHERE THEY ARE NOW DOING MY
DAUGHTERS UNNATURAL HISTORY
STUDY TRANSFORMED SINCE THE
'80s.
THESE ARE THINGS THAT NEED TO BE
BROUGHT INTO THE OPEN.
WE NEED DISCUSSION WITH THE
PARENTS AND WITH THE CAREGIVERS.
S ESPECIALLY FOR CHILDREN BRAIN
ARE NOT WORKING CORRECTLY AND
CANNOT SPEAK OR THINK FOR
THEMSELVES.
IT IS A SOCIETAL QUESTION.
THAT I THINK WE REALLY NEED TO
ADDRESS.
ONE LAST THING, MY RESEARCH
EMAILED US WORKING ON THE ARPA H
GETTING PRY YOUR PHASE FOR ARPA
H, HE SAID COLLEAGUES WERE
ASKING HIM WHY WOULD WE FOCUS ON
RARE DISEASES?
WHY WOULDN'T WE FOCUS ON SOME
MORE COMMON DISEASES?
AND WHAT ALL OF US DISCUSSED
AGAIN OFFLINE QUITE A BIT IS WE
WOULD KILL FOR CORTICOSTEROIDS
IF OUR KIDS HAD ASTHMA, KILL FOR
INSULIN PUMPS LIKE IF YOU ARE
DEALING WITH DIABETES, NOT TO
SAY WE CAN'T DO BETTER IN THOSE
SPACES BUT IF YOU CAN PERFORM
BASIC ACTIVITIES OF DAILY
LIVING, YOU HAVE GOT A HUGE STEP
UP.
SO I THINK WE CAN DO MORE WORK
TO TRY TO GET PEOPLE TO THAT
PLACE WHERE THEY CAN FUNCTION AT
EVEN BASIC LEVEL IN SOCIETY AND
THAT'S THE HOPE THE SCIENCE AND
NCATS IS BRINGING TO US BUT I
REALLY DO THINK BENEED TO HAVE
NCATS PULL IN, YOU HAVE BEEN
GOOD AT THIS, THE PATIENT
CAREGIVER COMMUNITIES, MAKE SURE
FDA IS REALLY LISTENING, MAKE
SURE CMS IS REALLY LISTENING THE
WHITE HOUSE IS LISTENING, AND
THAT WE ARE MOVING THIS FIELD
FORWARD AND WE ARE TAKING INTO
ACCOUNT THAT PERSPECTIVE WHEN
THINKING ABOUT RISK.
ONE LAST THING, IN TERMS OF THE
DATA DEMOCRATIZATION, I AGREE
THAT IS SO CRITICAL.
AND I'M THRILLED MY DAUGHTERS
DISEASE DAY IS PARTICIPATING IN
RARE X.
THE DATA IS OWN AND AVAILABLE
AND PATIENTS AND THEIR FAMILIES
MAKE THE DECISION TO SHARE IT
AND EVENTUALLY, I DON'T WANT TO
PUT KELLY ON THE SPOT BUT SHE'S
BEEN LEADING A LOT IN MAKING
SURE,IOTONLY THAT PATIENTS OWN
THEIR DATA BUT THAT THEY GET
COMPENSATED FOR THEIR DATA.
THAT IS IMPORTANT GOING TO THE
FUTURE.
WE KNOW THE STORY OF HENRY
LACKS, IF THE PATIENTS
THEMSELVES ARE THE ONES
GENERATING THESE MILLIONS AND
BILLIONS OF DOLLARS THAT SOME OF
THOSE RESOURCES ARE COMING BACK
FOR WHAT THEY PUT IN.
THAT IT SEEMS TO ME TWO CENTS,
HAPPY TO SHARE THE ARPA H WHITE
PAPER BUT I THINK NCATS IS
WELL-POSITIONED TO KEEP LEADING
THIS DISCUSSION AND MOVING IT
INTO THE 21ST CENTURY AND
HOPEFULLY WILL HAVE 21ST SENT
ARE I TREATMENTS AND CURES
HITTING THE MARKET AND AVAILABLE
FOR KIDS LIKE MY DAUGHTER AND
MORE.
THANK YOU.
>> THANK YOU SO MUCH, REINFORCES
WHY WE ARE GOING TO MISS YOU SO
MUCH ON COUNCIL.
HUE FOR JOINING US TODAY.
VERY WELL SAID.
ALL OF IT WAS WELL SAID AND WE
DO HAVE TINA MORRISON HERE, I
DON'T WANT IF YOU WANT TO
RESPOND QUICKLY, YOU HAVE A
COMMENT IN THE BOX.
DO YOU WANT TO SAY SOMETHING AND
UNMUTE YOURSELF?
>> THANKS FOR THE CHANCE, I
APPRECIATE IT.
CAN YOU HEAR ME OKAY?
>> WE CAN.
YES.
>> I AM GOING TO SHARE A LINK
FROM THE RECENT SUMMIT SPONSORED
BY THE CENTERS OF EXCELLENCE AND
REGULATORY SCIENCE AND
INNOVATION.
THERE WAS A REALLY IMPORTANT
PANEL DISCUSSION WITH FIVE FDA
COMMISSIONERS THE CURRENT ACTING
COMMISSIONER MODERATED THE
SESSION JANET WOODCOCK AND ONE
FASCINATING PIECE OF THE
DISCUSSION WAS FOCUSING ON RARE
DISEASE, TALKING ABOUT OUR
RELATIONSHIP WITH CMS, AND
PAYERS TO REALLY THINK ABOUT
WHAT STRUCK ME JONI IN YOUR
COMMENTS ABOUT GETTING MORE
TREATMENT TO ALL PATIENTS, OR
ALL PEOPLE, I FORGET YOUR EXACT
VISION BUT THE THING THAT CAME
TO MY MIND IS WE CAN DEVELOP THE
TREATMENTS BUT WHAT IS THE ROLE
OF NCATS AND HOW TO GET TO THE
PATIENT AND A LOT OF THAT HAS TO
DO WITH PAYERS AND CONVERSATION
WITH START WITH CMS AND JUST
THINKING THE ROLE FDA CAN PLAY
IN HELPING THAT AND I KNOW THAT
FDA HAS WORKED MY PAST
EXPERIENCES COME FROM
(INAUDIBLE) I WAS A REGULATOR
THERE FOR SEVERAL YEARS.
WE CONSTANTLY TALKED ABOUT THIS
CONVERSATION THAT NEEDS TO
HAPPEN BETWEEN FDA AND CMS AS A
START TO SAY WHAT IS THE DATA
FDA NEEDS?
WHAT IS THE DATA CMS NEEDS?
IS THERE WAY TO WORK TOGETHER SO
THAT ONCE SOMETHING LEAVES FDA
AND LANDS ON CMS DESK IS THERE A
WAY FOR US TO ANSWER THOSE
QUESTIONS FOR THEM IN THAT
PASSING OF THE BATON WHERE WE
WORK TOGETHER WHEN BATON IS
PASSED AND WE SUPPORT CMS
MISSION TO MAKE DECISIONS FASTER
BECAUSE SOMETIMES THAT IS A HUGE
HURDLE EVEN THE TREATMENTS ARE
THERE PATIENTS STILL CAN'T GET
THEM.
>> THANK YOU SO MUCH FOR THOSE
COMMENTS.
I COULDN'T AGREE MORE, THE GOAL
OR THE THREE GOALS I OUTLINE IN
MANTRA OF MORE TREATMENT TO ALL
PEOPLE MORE QUICKLY, THAT IS
NCATS CAN'T DO THAT ALONE.
IT IS ABOUT THE ENTIRE
BIOMEDICAL SYSTEM COMING
TOGETHER AND THE PATIENT AND
PATIENT ADVOCACY GROUPS COMING
TOGETHER.
KRISTINA, I APPRECIATE YOUR THE
WAY YOU CAN ARTICULATE THESE
THINGS BECAUSE YOU ARE A RARE
MOM AND YOU SEE THESE FIRSTHAND
AND I THINK THAT IS VALUABLE TO
BRING INTO THESE DISCUSSIONS.
SO THAT WE CAN FIGHT THOSE --
THE OPERATIONAL PIECE OF THIS
ARE SO IMPORTANT AND CRITICAL
FOR US TO ADDRESS THE AT THE
SAME TIME TIME WE ADDRESS
SCIENCE.
I.
A A RARE KID I GUESS.
I HAVEN'T THOUGHT ABOUT THAT HOW
TO SAY THAT BUT HOW YOU PHRASE
IT AS BEING A RARE MOM, IS SO
COMPELLING.
MY MOM HAD A RARE DISEASE, IT
WASN'T A GENETIC DISEASE, IT WAS
MORE DIFFERENT KINDS OF CANCER
RELATED CONDITIONS BUT IT WAS A
RARE DISEASE NONETHELESS SO
THESE KINDS OF ISSUES ARE JUST
REALLY IMPORTANT TO ME AND THE
MORE WE BRING THESE PEOPLE AND
COMMUNITIES TOGETHER OUR VOICES
ARE GOING TO BE LOUDER AND
LOUDER.
AND WILL CREATE THAT SWELL OF
OPPORTUNITIES COMMUNITY SO THAT
WE CAN BE SUCCESSFUL FOR THESE
GOALS.
I THINK UNFORTUNATELY I HAVE TO
CUT OFF OUR DISCUSSION BECAUSE
WE DO HAVE OUR SPECIAL GUEST WHO
HAS ARRIVED.
TARA SCHWETZ, I HOPE YOU ARE
ABLE TO BE ON SCREEN HERE.
I WANT TO QUICKLY INTRODUCE TARA
TARA.
FOR MUCH OF 2021 LAST YEAR TARA
SCHWETZ HAS BEEN ON DETAIL TO
WHITE HOUSE OFFICE OF SCIENCE
TECHNOLOGY AND POLICY AS THE
ASSISTANT DIRECTOR FOR
BIOMEDICAL SCIENCE INITIATIVES.
IN THIS ROLE SHE'S LED THE
EFFORTS TO STAND UP THE ADVANCE
RESEARCH PROJECTS AGENCY HEALTH
FOR ARPA H AND SHE'S BEEN
WORKING WITHIN THE BIDEN
ADMINISTRATION TO PROPOSE ASH PA
H TO TACKLE SOME OF THESE ISSUES
THAT WE HAVE BEEN TALKING ABOUT
AS WELL.
NOW SHE IS IN HER NEW ROLE AS
ACTING PRINCIPAL DEPUTY DIRECTOR
AT THE NIH SO WE ARE HAPPY TO
HAVE TARA BACK AT THE NIH, BUT
HER CONNECTIONS WITH THIS
PARTICULAR ARPA H OSTP LIAISON
AND DETAILING HAS -- I THINK
WILL PROVE VERY BENEFICIAL AS WE
GO FORWARD NO MATTER WHAT THAT
MIGHT LOOK LIKE.
SO TARA, I WILL TURN IT TO YOU
AND HAVE YOU GIVE US A STATUS
UPDATE ON WHAT YOU KNOW ABOUT
ARPA H.
THANK YOU SO MUCH FOR JOINING
US.
>> THAW FOR HAVING ME.
I HOPE YOU CAN HEAR ME.
I'M GOING TO PREFACE THIS I HAVE
BEEN HAVING INTERNET TROUBLE ALL
DAY ON AND OFF SO I HAVE BACK
UPON BACK UP HERE IF I NEED TO
SWITCH NETWORKS AND COMPUTERS
AND EVERYTHING ELSE.
I'M GLAD I WAS ABLE TO GET ON
COUPLE OF MINUTES EARLY TO HEAR
THE END OF THAT DISCUSSION
BECAUSE I THINK THIS TIES SO
NICELY WITH WHAT SOME OF THE
ROLES THAT ARPA H CAN PLAY
PARTICULARLY TO THE LAST
COMMENTS BECAUSE I HOPE ARPA H
IS A FACILITATOR BETWEEN THE
RESEARCH EFFORTS COMING OUT
WORKING WITH FDA AND CMS MORE
CLOSELY, THAT WILL BE A CRITICAL
ROLE: SO WE CAN HELP ADVANCE
THAT AS ARPA H HOPEFULLY GETS
PULLED TOGETHER AND IS ABLE GET
LAUNCHED.
I KNOW Y'ALL HEARD ABOUT ARPA H
BEFORE AND CLEARLY BY DISCUSSION
THIS IS NOTHING COMPLETELY NEW
FOR ALL OF YOU BUT JUST TO SORT
OF TAKE A STEP BACK AND ENSURE
EVERYONE IS ON THE SAME PAGE, I
WANT TO REMIND FOLKS ARPA H IS
ADVANCE RESEARCH PRODUCT AGENCY
FOR HEALTH.
THIS IS BEING PROPOSED AS A NEW
ENTITY THAT WILL EQUITABLY
BENEFIT HEALTH OF ALL AMERICANS
BY HELPING CATALYZE FULL BREAK
THROUGH, THE SHORT AND SIMPLE OF
IT.
IF YOU CAN GO TO THE NEXT SLIDE.
THINKING ABOUT WHAT COULD BE
DONE AND WHAT HAS BEEN DONE OVER
THE LAST SEVERAL DECADES.
WE KNOW SCIENCE HAS DONE AMAZING
THINGS.
WE ARE AT THIS REALLY
UNPRECEDENTED MOMENT OF PROGRESS
PARTICULARLY AS WE ARE STILL
MISPANDEMIC BUT WE HAVE BEEN
ABLE THE SEE SOME AMAZING
SCIENTIFIC WORK COME OUT OF WORK
DONE AND SUPPORTED BY NCATS BUT
IT TAKES CAUSES TAKE A STEP BACK
AND SAY WHAT MORE CAN WE DO TO
SPEED REVOLUTIONIZE AND
TRANSFORM MEDICINE AND IMPROVE
THE HEALTH OF ALL AMERICANS.
SO IF WE IMAGINE FOR -- FOR
INSTANCE, IF WE CAN PROGRAM
MRNA TO PRE-VACCINATE US
AGAINST 50 MOST COMMON MUTATIONS
TA DRIVE CANCER, THIS GIVES OUR
BODIES A CHANCE TO TARGET AND
ELIMINATE BEFORE THEY CAN CREATE
A TUMOR, WHAT IF WE COULD
DEVELOP DRUGS THAT HAVE PRECISE
MOLECULAR ZIP CODES THAT WOULD
ALLOW FOR UNPRECEDENTED
TARGETING ACROSS THE BLOOD BRAIN
BARRIER TO SPECIFIC CELLS IN OUR
BODY, WITHOUT ANY UNTOWARD SIDE
EFFECTS AND THESE ARE JUST
COUPLE OF EXAMPLES AND THERE ARE
OTHERS ON THE SLIDE AS WELL OF
THE POTENTIAL CAPABILITIES AND
PLATFORMS THAT COULD BE POSSIBLE
IF WE COULD HARNESS THE BEST
IDEAS AT THE RIGHT TIME WITH THE
RIGHT AMOUNT OF RESOURCES.
I'M SURE Y'ALL HAVE BETTER IDEAS
YOU ARE HIKING AS YOU LOOK AT
THIS SLIDE.
I THINK THAT FOR ME WHAT THAT
SIGNAL IS POTENTIAL FOR
OPPORTUNITIES HERE THAT WE HAVE
WITH ARPA H IS EVEN MORE
EXCITING AND REALLY TREMENDOUS.
SO WE MOVE TO THE NEXT SLIDE.
I JUST WANT TO QUICKLY GO OVER
WHAT POTENTIAL VERSION OF THE
MISSION COULD READ BECAUSE OF
COURSE AT MOMENT ARPA H IS STILL
JUST A PROPOSAL.
BUT WHAT WE HAVE HERE IS THAT
AID COULD BENEFIT THE HEALTH OF
ALL AMERICANS BY UTILIZING
HEALTH BREAK THROUGHS NOT
READILY ACCOMPLISHED THROUGH
TRADITIONAL RESEARCH OR
COMMERCIAL ACTIVITY.
TO ME THE LAST POINT IS REALLY
KEY BECAUSE WHAT WE ARE TALKING
ABOUT IS DOING THINGS THAT CAN'T
BE DONE CURRENTLY, THAT ARE
REALLY INCREDIBLY DIFFICULT TO
DO.
THAT IS THE SPACE THAT ARPA H
WANTS TO LIVE IN.
ADVANCING THIS MISSION AND THE
IDEAS MENTIONED PREVIOUSLY, IS
GOING TO REQUIRE A NOVEL
APPROACH TO MORE SUPPORTING
BROAD BIOMEDICAL RESEARCH.
AND THE IDEA WITH ARPA H IS TO
LEVERAGE --
>> WE HAVE LOST YOUR AUDIO.
HOPEFULLY YOU CAN STILL HEAR US?
>> I CAN.
>> I HEAR TARA JUST FINE.
HAVE OTHERS?
>> SAME HERE.
>> I'M HEARING HER FINE AS WELL.
>> WE CAN HEAR HER.
>> OKAY.
GOOD.
I WAS WORRIED FOR A SECOND.
I HAVE OTHER DEVICES TO SWITCH
TO IF NEEDED.
HOPEFULLY AUDIO GETS CORRECTED
ON YOUR END AND OTHERS CAN STILL
HEAR ME.
TO GO BACK TO THIS APPROACH,
THAT ARPA H IS GOING TO TAKE,
LEVERAGING WHAT WAS PIONEERED AT
DARPA, IT'S UTILIZED THERE FOR
60 PLUS YEARS, AND HAS ALSO BEEN
APPLIED IN OTHER AREAS OF R&D
MORE RECENTLY.
IF YOU THINK ABOUT ARPA E, AND
EVEN BRTA, AND WE REALLY WANT TO
EMBRACE THIS APPROACH FOR
BIOMEDICAL AND HEALTH RESEARCH.
WE PROPOSE TO CREATE THIS
DISTINCT ENTITY WITHIN NIH
THAT'S HAVE AUTONOMY AND
INDEPENDENCE RESOURCES AND
AUTHORITIES TO TACKLE SOME OF
THESE BIG CHALLENGES FACING
HUMAN HEALTH.
IN THE NEXT SLIDE WE TALK ABOUT
THIS ARPA MODEL.
FOR ANYONE WHO MAY BE LESS
FAMILIAR JUST AGAIN TO LEVEL A
LITTLE, THE MODEL EMBRACES A
PROGRAM MANAGER CENTRIC
MENTALITY, IT PROMOTES ACTIVE
PROJECT MANAGEMENT AND
ACCOUNTABILITY THROUGH
QUANTIFIABLE METRICS AN STAGE
GATING.
IT APPROACH THE ORGANIZATION
THROUGH A LENS OF BEING LEAN AND
MINIMUMMABLE BUT BUILDS IN A
FAIR AMOUNT OF AUTONOMY AND
ACCOUNTABILITY.
IT FOCUSES ON MISSION DRIVEN
RATHER THAN REQUIREMENT OR CAST
DRIVEN -- TASK DRIVEN RESEARCH.
MENT THAT WHICH IS BOLD AND
RISKY BUT COULD OFFER REALLY
HIGH RETURN.
T THAT ADHERES TO URGENT TIME
BOUND PRINCIPLES THAT ARE
APPLIED NOT JUST TO THE PROGRAM
-- BUT ALSO TO THE STAFF TENURE.
IN REFERENCE TO SORT OF THIS
HIGH RISK HIGH REWARD APPROACH I
LIKE TO GIVE THE ANALOGY THAT
WHAT WE WANT TO DO WITH ARPA H
IS ON SOME LEVEL, HITTING -- IT
IS LIKE THROWING A DART IN A
DARK ROOM.
IF YOU HIT A DARK DART BOARD, ON
THE BULLS EYE IT IS AMAZING.
AND IF YOU DON'T YOU LEARN THAT
IT IS NOT IN THAT LOCATION AND
NOW YOU CAN AIM SOMEWHERE ELSE.
BUT OBVIOUSLY BUILDING OFF AND
LEARNING FROM WHAT YOU ARE DOING
BUT HAVING THE POTENTIAL TO HAVE
A REALLY HIGH IMPACT SO IF WE
MOVE TO THE NEXT SLIDE.
TO ENUMERATE THE GOALS OF ARPA
H, POTENTIAL GOALS OF ARPA H.
THEY ARE HERE SUPPORTING
TRANSFORMATIVE HIGH RISK HIGH
REWARD RESEARCH OF COURSE BUT
DOING SO BY EMBRACING A SENSE OF
URGENCY AND SPEEDING THE
APPLICATION IMPLEMENTATION OF
BREAK THROUGHS AT VARIETY OF
DIFFERENT LEVELS.
SO WE SAY FROM THE MOLECULAR TO
THE SOCIETAL.
AND RECOGNIZING THAT IS PRETTY
BROAD BUT DOING SO IN A WAY THEY
SERVE ALL PEOPLE.
TO BUILD CAPABILITIES AND
PLATFORMS THAT ARE BROADLY
APPLICABLE ACROSS A WIDE RANGE
OF DISEASE AND CONDITIONS.
TO REVOLUTIONIZE HOW WE PREVENT
DETECT TREAT CURE THEM.
WE WANT TO FOCUS ON CONVERTING
USE DRIVEN IDEAS SO THOSE IDEAS
WITH DIRECT APPLICATION INTO THE
SOLUTION FOR PATIENTS REALLY
QUICKLY.
AND THEN HELP OVERCOME MARKET
FAILURES THROUGH CREATIVE
SOLUTIONS AND DERISKING.
THERE ARE AREAS RIPE FOR
TRANSFORMATION IF STIMULATED
THEM THROUGH THE RIGHT SUPPORT
AND COLLABORATION.
IF WE MOVE TO THE NEXT SLIDE.
THE GUIDING PRINCIPLES THAT
DRIVE ARPA H ECHO ELEMENTS OF
THE ARPA MODEL.
ARPA H IS CENTERED AROUND AND
SHOWING THIS RISK TOLERANCE OR
RISK ACCEPTANCE, AND INSTILLING
KEY FOUNDATIONAL VALUES THAT ARE
INHERENT IN THE ARPA MODEL.
BUT IN ORDER TO DO THAT WE
RECOGNIZE CULTURE IS KEY TO ARPA
H SUCCESS.
THAT IS SOMETHING NCATS WORKED
HARD TO BUILD AND DEVELOP OVER
ITS TEN PLUS YEARS NOW HISTORY.
AND THINKING ABOUT WHERE ARPA H
SHOULD LIVE, THE ADMINISTRATION
SUPPORTS ARPA H, HE PROPOSES A
COME PENT OF NIH.
AND LINKED TO NIH REALLY DRAW ON
THE VAST KNOWLEDGE EXPERTISE AND
INFRASTRUCTURE THAT CURRENTLY
EXISTS HERE AT NIH.
INCLUDING INSTITUTES LIKE NCATS
AND THINKING CONNECTIONS AND
COLLABORATIONS AND SYNERGIES
THAT WE CAN BUILD THERE.
BUT RECOGNIZING THAT IT DOES
NEED TO BE DISTINCT AND HAVE ITS
OWN CULTURE AND ORGANIZATION
AUTONOMY.
IN DOING SO ARPA H IS GOING TO
SEEK THE BRING ON THE MOST
INNOVATIVE PEOPLE WITH THE MOST
NOVEL IDEAS THAT WILL FORM
ROBUSTLY DIVERSE AND
COLLABORATIVE TEAM.
AND EQUITY IS GOING TO BE
ESSENTIAL AND GOING TO BE
CONSIDERED ESSENTIAL ELEMENT OF
ARPA H AND KEY IN ITS INTERNAL
PRACTICES LIKE HIRING AND IN THE
PROJECTS AND PROGRAMS IT
SUPPORTS, I LOOK AT THIS IN SORT
OF THREE WAYS.
RIGHT?
THINKING ABOUT ROBUST EQUITY IN
THE PEOPLE THAT HIRE, THE
PROGRAMS THAT SUPPORT, AND THE
PERFORMERS IT MAKES AWARDS TO.
CONTINUING WHICH IS DOES AROUND
PEOPLE, THE DIRECTOR OF ARPA H
AS WELL AS PROGRAM MANAGERS ARE
INTENDED TO BE TERM LIMITED AND
NOT A 20 YEAR TERM, MUCH SHORTER
TERMS.
THINKING IN THE LIKE THREE TO
FIVE YEAR RANGE.
THIS FIRST DIRECTOR, THEY ARE
GOING TO SET THE STAGE FOR SO
MANY YEARS TO COME AND IT IS
GOING TO BE REALLY CRITICAL FOR
US TO FIND THE RIGHT LEADER,
THAT'S GOING TO EXCEL
TECHNICALLY AND AS A LEADER.
IT WILL ALSO BE A REALLY FLAT
DYNAMIC ORGANIZATION BUILT
NIMBLE AND OPERATIONALIZING
SCIENTIFIC PORTFOLIO.
AND BUSINESS PROCESSES.
AGAIN WE TALK ABOUT THAT SENSE
OF URGENCY AND OPERATING UNDER
TIME BOUND PRINCIPLES.
WHAT WE MEAN BY THAT IN ADDITION
TO TERM LIMITS WHAT I ALWAYS
HEAR WHAT I HAVE HEARD MANY,
MANY TIMES FROM THE DRPA FOLKS I
HAVE SPOKEN WITH IS WHEN THEY
COME ON BOARD, THEY GET THEIR
BADGE AND THEIR BADGE HAS AN
EXPIRATION DATE ON IT AND THAT'S
THEIR TICKING TIME CLOCK.
THEY LOOK AT THAT EVERY DAY WHEN
THEY PLUG TO COMPUTER OR WHEN WE
WERE GOING BACK TO THE OFFICE
AND THEY SCANNED IT TO GET IN
THE BUILDING OR THROUGH THE GATE
OR WHATEVER.
IT WAS A CONSTANT REMINDER OF
WHAT THEY WANTED TO ACHIEVE OVER
THAT TIME PERIOD.
I THINK THAT THAT IS JUST A
REALLY NICE MANIFESTATION OF
LIKE THAT CULTURAL ASPECT.
I WILL ALSO MENTION IN TERMS OF
THINKING THROUGH TIME BOUND
PRINCIPLES.
IT IS GOING TO BE GOING TO
OPERATE WITH UTILIZING DIFFERENT
PROCESSES.
FROM THE REST OF NIH, IT LIKELY
WILL NOT BE ISSUING GRANTS BUT
MORE TAKE AGO APPROPRIATE OF
CONTRACTS AILINGOR AND
TRANSACTION AND CHALLENGES,
THINGS YOU ARE FAMILIAR WITH BUT
WANTED TO MENTION THAT.
WITHOUT DWELL ON THIS SLIDE TOO
MUCH LONGER, I JUST WANT TO
REITERATE THE IMPORTANCE OF OPEN
AND TRANSPARENT.
ABOUT PROsS AND APPROACHES AS
IT DEVELOPS THEM.
AND IMPORTANTLY, ENGAGING
STAKEHOLDERS ACROSS A WIDE RANGE
OF SECTORS AND DISCIPLINES EARLY
AND OFTEN.
IF WE ARE DEVELOPING THIS AS WE
TALK ABOUT IT WITH A FOCUS ON
USE DRIVEN RESEARCH, WE WANT TO
BE COGNIZANT ABOUT THE FACT THAT
THOSE END USERS SHOULD BE
INVOLVED AND AT THE TABLE FROM
THE BEGINNING STAGES OF
DEVELOPING A PROGRAM.
AND CONSULTING THROUGH THE
PROCESS.
AND TO MISQUOTE I GUESS, THE --
I GUESS OFTEN USED FIELD OF
DREAMS ANALOGY, IF YOU BUILD IT
THEY WILL COME, WHAT WE DON'T
WANT TO DO IS BUILD IT, HAVE
THEM COME AND THEN WHY ON EARTH
DID YOU BUILD A BASEBALL PEELED
IN THE MIDDLE OF A CORN FIELD?
AND SOMETIMES THAT WORKS AND IT
IS AMAZING BUT OTHER TIMES I ASK
WHY DIDN'T YOU BUILD A SOCCER
FIELD, THAT'S WHAT WE WOULD HAVE
WANTED.
SOMETHING COMPLETELY DIFFERENT.
NOT EVEN A FIELD TO PLAY A
SUPPORT ON SO JUST RECOGNIZING
THE IMPORTANCE OF THAT REALLY
EARLY AND FREQUENT ENGAGEMENT.
AND PROMOTING INDEPENDENCE AND
ACCOUNTABILITY THAT REINFORCED
THROUGH AMBITIOUS MILESTONES AND
METRICS.
THIS WILL BE DRIVEN THROUGH THE
PROGRAM MANAGERS PRIMARILY.
WHO WILL HAVE BROAD AUTONOMY AND
AUTHORITY TO BE CREATIVE AND
DRIVE INVASION AND TRACK METRICS
AND MAKE DECISIONS ABOUT FUTURES
OF THE PROGRAM BASED ON THOSE
METRICS.
THEN OF COURSE FOLLOWING A FAIL
FORWARD APPROACH AND EMBRACING
THE ETHOS OF IF YOU ARE GOING TO
FAIL, FAIL EARLY AND ACCEPTING
FAILURE AS A NORMAL EXPECTED
COMPONENT OF ITS OPERATIONS SO
ESSENTIALLY WHAT THIS MEANS IS
YOU ARE FAILING IF YOU ARE NOT
AT LEAST FAILING OCCASIONALLY.
IF WE CAN MOVE TO NEXT SLIDE.
I WILL CALL OUT OUT AUTHORITIES
NEEDED TO DO THIS BECAUSE THIS
IS REALLY KEY AND CRITICAL TO
SUCCESS.
MOSTLY A LOT OF THEM ANYWAY ARE
AROUND ABILITY TO HIRE AND
RECRUIT THE BEST PEOPLE.
AND HAVING FLEXIBLE FUNDING
AUTHORITIES THAT ALLOW FOR USE
OF MULTIPLE APPROACH TO
DISTRIBUTE RESOURCES.
AND TO DO SO OVER MULTIPLE
YEARS.
SO AGAIN THIS WOULD BE THINGS
LIKE OTHER TRANSACTION AND PRIZE
AUTHORITIES AND MULTI-YEAR
APPROPRIATIONS.
THE PEER REVIEW PROCESS ARE
ROBUST AND EXPERT DRIVEN BUT
DIFFERENT THAN TRADITIONAL NIH
PEER REVIEW PROCESS AND
AUTHORITIES WOULD BE IMMEDIATED
TO ENSURE THIS IS POSSIBLE FOR
ALL MECHANISMS ESPECIALLY IF
PLACED WITHIN NIH
INFRASTRUCTURE.
THERE ARE OBVIOUSLY A FEW OTHER
THINGS LIKE EXEMPTING CERTAIN
INFORMATION FROM FOIA, RELATED
TO COMPANIES BUSINESS AND
COMMERCIALIZATION INVESTMENT
PLANS AND COLLABORATING WITH AND
POTENTIALLY REIMBURSING FDA FOR
CERTAIN ACTIVITIES.
AND THIS WOULD BE A WAY TO
FACILITATE THAT INTERACTION IN A
MUCH MORE ROBUST AND DEFINED
WAY.
IF WE MOVE TO THE NEXT SLIDE.
WE ARE ASKED QUITE A BIT ABOUT
WHERE IDEAS WILL COME FROM.
SO HERE JUST TRY TO ILLUSTRATE
THAT IDEAS ARE GOING TO COME
FROM AND FORMED BY MANY
DIFFERENT SOURCES.
PATIENTS ADVOCATES, ACADEMIA,
INDUSTRY AS WELL AS OTHER
SCIENTIFIC SAKE HOLDERS LIKE
FROM ACROSS THE FEDERAL
GOVERNMENT AND IDEAS GENERATED
AND REFINED BY CONSIDERING THE
DIVERSE ARRAY OF PERSPECTIVES,
BRINGING IN PEOPLE WITH
DIFFERENT BACKGROUNDS AND
EXPERTISE.
DIFFERENT STAKEHOLDERS,
DIFFERENT SCIENTIFIC
DISCIPLINES, DIFFERENT
PARTNERSHIPS AND MORE.
THE ARPA H DIRECTOR HAS AUTONOMY
TO STEP PRIORITIES AND PROVIDE
PROGRAM MANAGER WHOSE WILL COME
BEARING IDEAS WITH THE
INDEPENDENCE TO ENACT THAT
VISION.
WITH ROBUST SUPPORT BUT OFTEN
MINIMAL INVOLVEMENT FROM THE
DIRECTOR.
ESSENTIALLY WHAT THAT MEANS IS
HIRE REALLY GOOD SMART PEOPLE
AND GIVE THEM THE FREEDOM TO DO
WHAT THEY DO BEST.
THAT'S WHAT ARPA H PROGRAM
MANAGERS WILL BE DOING, THEY
WILL BE RESPONSIBLE FOR TAKING
IN FEEDBACK FROM ALL THESE
DIFFERENT SOURCES, THAT THEY ARE
RECEIVING.
AND DRAFTING OLD PROGRAMS NEXT
SLIDE WE DON'T HAVE THE GOOD
IDEAS AND KNOW CHALLENGES AND
ISSUES FACING THE COMMUNITY WE
HAVE BEEN TRYING TO FIRST
FOREMOST SHARE INFORMATION ABOUT
ARPA H AS AVAILABLE ALSO MORE
IMPORTANTLY TO HEAR FROM THE
COMMUNITY.
TO DATE NIH AND OSTP HELD AN
INFORMATION SESSION AVAILABLE
ONLINE, PUBLISHED COMMENTARY IN
SCIENCE AND HAS HELD NUMEROUS
MEETINGS WITH DIFFERENT
ORGANIZATIONS, VARIETY OF
DIFFERENT PRESENTATIONS, SUCH AS
THIS.
WE ALSO UNDERTOOK A SERIES OF
LISTENING SESSIONS.
TO HEAR FROM DIFFERENT VOICES
ACROSS THE COMMUNITY.
GO TO NEXT SLIDE SUMMARIZE THOSE
LISTENING SESSIONS WHICH WERE
LED BY LEADERSHIP FROM ACROSS
THE OSPTP AND NIH INCLUDE ALL 27
INSTITUTE AND CENTER DIRECTORS
AND JONI PARTICIPATED IN ONE OF
THOSE.
OVER THE COURSE OF WE HAD 15
SESSIONS AND THEN ONE FEEDBACK
SESSION, 16.
WE HEARD FROM OVER 5100
STAKEHOLDERS INCLUDING NEARLY
250 ORGANIZATIONS FROM ALL OVER
THE COUNTRY.
I HOPE MANY OF YOU ARE ABLE TO
PARTICIPATE IN THOSE.
WHAT CAME OUT OF THAT IS SEVERAL
THEMES THAT WE HAVE GROUPED INTO
TWO MAIN BUCKETS.
COMMENTS ON THE NATURE OF
SCIENTIFIC PORTFOLIO AND THE KEY
ASPECT ON WHICH TO FOCUS AS WELL
AS THE ADOPTION OF STREAMLINE
YET NIMBLE PROCESSES FOR
FLEXIBILITY AND BROAD INCLUSION.
I FOR GOT TO SAY -- HERE IS AN
ANIMATION HERE BUT IF WE CAN
MOVE FORWARD TWO NOW.
THAT WOULD BE GOOD BECAUSE I
WILL TALK ABOUT THAT A LITTLE
BIT FURTHER.
SO IF WE MOVE TO THE NEXT SLIDE.
THE AREAS OF FOCUS ON THE
PORTFOLIO IN TERMS OF FEEDBACK
WE RECEIVE REALLY CENTERED
AROUND THE NEED TO COMPLIMENT
NIH RESEARCH PORTFOLIO AND NOT
DUPLICATE IT AND OF COURSE THAT
SOMETHING THAT WE OBVIOUSLY ARE
VERY KEENLY AWARE OF AND WANT TO
TAKE STEPS TO AVOID.
AND REALLY TO ALSO NOT JUST
AVOID DUPLICATION BUT IDENTIFY
AREAS FOR COLLABORATION AND
SYNERGY TO ENHANCE EFFORTS
ACROSS BOTH ORGANIZATIONS
WHETHER ARPA H OR NIH INSTITUTES
AND CENTERS.
WE ALSO HEARD ROBUSTLY ABOUT THE
IMPORTANCE AND CRITICALITY OF
ADDRESSING HEALTH INEQUITIES.
AND PROMOTING HEALTH EQUITY AND
THIS IS IMPORTANT TO THE
ADMINISTRATION BROADLY AND
SOMETHING WE IN THINKING ABOUT
SETTING UP A NEW ORGANIZATION WE
WANT TO BUILD IN TO IT FROM ITS
FOUNDATION.
WE HEARD A DESIRE TO HAVE -- TO
BUILD TECHNOLOGIES, PLATFORMS
THAT ARE REALLY BROADLY
APPLICABLE THAT ARE NOT DISEASE
SPECIFIC.
ALSO WE HEARD BROADLY ABOUT
EVERY SINGLE COMMENT YOU CAN
IMAGINE ABOUT DATA SO THINKING
ABOUT ACCESSIBILITY INTEGRATION
SHARING STANDARDS TOOLS, ET
CETERA.
HOW TO LEVERAGE AI IN CREATIVE
AND UNIQUE WAYS.
AND FOCUS ON COMMERCIALIZATION
AND PUSHING PRODUCT CLOSER TO
MARK TO GET TO PATIENTS.
WE HEARD ABOUT PROCESSES.
AND THE MAIN TAKE HOME HERE IS
THE NEED THE STREAMLINE
PROCESSES TO EXPEDITE DISCOVERY
THROUGH INTEGRATION OF THE
COMMUNITY PATIENTS AND THEIR
PROVIDERS EARLY IN THE PROCESS
AS MENTIONED BEFORE.
BUT BUILDING MULTI-DISCIPLINARY
COLLABORATION AND PARTNERSHIPS,
SO DRAWING IN SCIENTISTS FROM
OTHER DISCIPLINES, HOW WE CAN
PULL IN FOLKS FROM CHEMISTRY OR
MATH OR PHYSICS.
ENGINEERING.
I KNOW THIS IS SOMETHING THAT
THERE IS A PUSH FOR THIS ACROSS
SCIENCE, THAT WAS EMPHASIZED WE
SHOULD FOCUS ON WITH ARPA H AND
WORKING WITH INDUSTRY
NON-PROFITS AND OTHERS IN THE
PRIVATE SECTOR.
SO GO TO NEXT SLIDE.
BACK IN SEPTEMBER WE RELEASED A
REPORT SUMMARIZING THE FEEDBACK,
THAT IS AVAILABLE ON LINE, ON
THE OSTP AND I BELIEVE ALSO
POSTED ON THE HIPPOWEBSITE.
AS I MENTIONED -- NIH WEBSITE.
WE HELD A FOLLOW-UP LISTENING
AND FEEDBACK SESSION TO ENSURE
THAT WHAT WE HEARD INDICATED TO
US WE CAPTURED IN OUR ELEMENTS
AND THAT WE WERE HEARING FOLKS
CORRECTLY.
WE ALSO HOOKED TOGETHER
ADDITIONAL FEEDBACK HOW TO
ENGAGE WITH THE COMMUNITY.
IF YOU MOVE TO THE NEXT SLIDE.
JUST SORT OF UPDATE I GUESS OR
REMINDER MAYBE WHERE THINGS
STAND.
THE PRESIDENT IN HIS FY 22
BUDGET REQUEST INCLUDED
$6.5 BILLION FOR ARPA H, NOW THE
HOUSE INCLUDED ARPA H IN THEIR
DRAFT FY 22 APPROPRIATIONS BILL
AND PUT FORTH 3 BILLION IN
APPROPRIATIONS.
WE SAW SIMILAR LANGUAGE ON THE
SENATE SIDE WITH BUDGET OF
2.4 BILLION SO OF COURSE THE FY
22 APPROPRIATION HASN'T
OFFICIALLY MOVED FORWARD OR
PASSED YET SO WE ARE KIND OF IN
A WAITING STATUS THERE TO SEE
HOW THIS ALL PLAYS OUT AND WHERE
IT ENDS UP GETTING FINALIZED IN
THE MIDDLE.
IF YOU MOVE TO NEXT SLIDE IN
TERMS OF OUR AUTHORIZATION,
THERE ARE COUPLE OF OPTIONS FOR
AUTHORIZATION THAT HAVE BEEN
INTRODUCED.
THERE WAS A BILL REPRESENTATIVE
ESCHEW RELEASED IN OCTOBER AND
THE CARES 2.0 BILL THAT
REPRESENTATIVES UPTON INTRODUCED
IN OCTOBER AND WE HOPE TO SEE
MOVEMENT SOON.
ONE THING I WILL SAY IS IN
ADDITION TO PRESIDENTIAL SUPPORT
THIS INITIATIVE HAS, WE'VE HEARD
GREAT BIPARTISAN CONGRESSIONAL
SUPPORT AND SO OPTIMISTIC ABOUT
IT, ITS SUCCESS FOR HOPEFULLY FY
22 BUT FOR THE FUTURE.
SO YOU GO TO THE FINAL SLIDE I
BELIEVE.
THE TAKE HOME HERE IS THAT WE
KNOW THIS ARPA MODEL WORKS.
AND WE HAVE SEEN HOW SUCCESSFUL
DARPA HAS BEEN.
THERE ARE SO MANY AREAS RIPE FOR
TRANSFORMATION.
WE JUST NEED TO HARNESS AND
FOSTER THEM SO THAT WE CAN TAKE
THESE POTENTIAL OPPORTUNITIES TO
SHAPE THE FUTURE OF HEALTH AND
MEDICINE AND SIGNIFICANTLY
IMPROVE QUALITY OF LIFE FOR ALL
AMERICANS BY LEVERAGING THIS
MODEL WE KNOW WORKS.
SO WITH THAT JONI, I DON'T KNOW
I THINK WE STILL HAVE A FEW
MINUTES OR QUESTIONS OR
COMMENTS?
>> THANK YOU SO MUCH.
REALLY APPRECIATE IT.
WE DO HAVE A FEW MINUTES FOR
QUESTIONS.
PAUL I SEE YOUR HAND FIRST
PLEASE GO AHEAD.
>> TARA GREAT JOB.
THANK YOU FOR THAT PRESENTATION.
WHERE DO YOU SEE OR WHERE DO THE
GROUP THINKING ABOUT THIS MOST
SEE THE IC RECORDS SITTING HERE?
I'M THINKING ONE LEVEL DOWN
MAKING SURE WE DON'T HAVE THAT
DUPLICATION OF EFFORT AND
FOCUSING ON THE RIGHT PROBLEMS.
>> OVER THE SUMMER AND INTO
FALL, THE NIH LEADERSHIP
CONVENED A GROUP OF IC DIRECTORS
THINK THROUGH HOW HAY WORK
TOGETHER AND I CAN'T RECALL JONI
IF YOU WERE ON THAT GROUP.
>> YES.
>> I KNOW I SPOKE AT THEM ONCE
OR TWICE.
THEY PROVIDED SOME REALLY GREAT
FEEDBACK IN THINKING THROUGH HOW
WE CAN COORDINATE, HOW WE COULD
ENSURE THAT THERE WAS AN
EXCHANGE OF IDEAS AND EXCHANGE
OF INFORMATION ACROSS THEM.
GOING FORWARD THERE'S A COUPLE
OF AREAS WE CAN ENOR ENVISION
COLLABORATION BETWEEN ARPA H AND
ICs AND THAT'S FIRST AND
FOREMOST REVIEW PROCESS WILL BE
MOSTLY HEARD OF FEDERAL DRIVEN
PROCESS AND WE'LL NEED EXPERT
REVIEWERS TO REVIEW SOME OF THE
ARPA H APPLICATIONS.
SO THAT TO ME IS A GREAT WAY TO
HAVE INVOLVEMENT AND TO LEVERAGE
EXPERTISE OF DIFFERENT ICs.
WE ALSO KNOW THERE'S GOING TO BE
SOURCES OF IDEAS AND SO SHOULD
-- GOING TO TRY TO THINK THROUGH
A FEEDBACK LOOP IF YOU WILL FOR
DEVELOPMENT OF THOSE IDEAS AND
WHAT EXACTLY THAT MEANS AND LOOK
LIKE.
AND I THINK WITH ARPA H
ESPECIALLY AS THIS IS GETTING
DEVELOPED, THIS IS NOT LIMITED
TO THE OTHER NIH ICs BUT
BROADER ACROSS THE FEDERAL
GOVERNMENT, WE WANT TO MAKE SURE
THAT PEOPLE ARE AWARE OF AND
SUPPORTIVE OF ARPA H AND WHAT IT
IS DOING AND TRYING TO THINK
THROUGH REALLY ROBUST WAYS TO
FACILITATE STAFF INTERACTION AND
EVEN DETAILS AND OTHER WORK KIND
OF EXCHANGE OPPORTUNITIES AS WE
CONTINUE TO BUILD UP THIS EFFORT
AND WE DISCUSSED THESE ALSO ON
THE INTERAGENCY LEVEL TOO
BECAUSE WE HAVE INTERAGENCY
COMMITTEE.
>> THANKS.
RAJESH YOU HAD YOUR HAND UP.
>> AS SOMEONE WHO SORT OF WORKED
INDUSTRY CAME TO NIH THEN LEFT
NIH GO BACK TO ACADEMIA AND
INDUSTRY, I WOULD RAISE A COUPLE
OF THINGS THAT I -- YOU MIGHT
HAVE HEARD ME TALK ABOUT.
THIS MODEL OF ESSENTIALLY HIRING
WORK FORCE THAT IS TERM LIMITED
IS RUNS COUNTER TO THE
COMPENSATION SCHEME AVAILABLE IN
THE GOVERNMENT.
SO IF YOU ARE PLANNING TO HIRE
THE BIGGEST BEST AND BRIGHTEST
INCLUDING THE DIRECTOR OF ARPA
H, YOU CAN PAY A PERSON THESE
PRESIDENT SALARY, NOT SURE YOU
ARE GOING TO GET A LIST
CHARACTERS.
SO I'M VERY SKEPTICAL ABOUT THAT
PARTICULAR PIECE OF HOW YOU WILL
STRUCTURE AND IF IT WILL SIT
WITHIN THE NIH.
THE OTHER PART I GUESS LINKED TO
THE IC QUESTION, WILL THIS BE AN
IC, WILL THIS BE SAME SANDING OF
IC, WILL IT BE OFFICE?
WHAT IS IT STRUCTURE?
I WAS INVOLVED IN THE STANDING
UP OF NCATS, THERE IS A 19
WHATEVER -- 79 OR 89 LAW, 26
INSTITUTES AT NIH.
SO IF ONE NEEDS TO CREATE A NEW
ONE, ONE NEEDS TO DISAPPEAR.
LAST TIME IT WAS NCRR TO CREATE
NCATS.
SO THAT'S BEGS THE QUESTION TO
WHAT LEVEL THIS IS BEING SET UP.
LINKED TO THAT QUESTION IS NCATS
COUNCIL MEMBER, I GUESS I WOULD
JUST RAISE THE PRESENTATION AND
VALUES YOU ESPOUSED AND ETHOS
YOU WANT THERE, THESE ARE THINGS
THAT WE ARE SEEING IN PRACTICE
BEING PUT MANY PLAY BY THE NCATS
STAFF.
SO HOW SHALL I PUT IT, A
VOLKSWAGEN AND AUDI ARE ON THE
SAME CHASSI.
SO IS CAMRY AND INFINITY.
SO IF THERE IS A REQUIREMENT FOR
REBRANDING THAT WOULD MAKE A
DIFFERENCE OKAY WE CAN SEPTEMBER
THAT BUT YOU GOT A FRAMEWORK --
ACCEPT THAT, BUT YOU HAVE A
FRAMEWORK OF SOMETHING WHY NOT
USE WHAT YOU HAVE TO BUILD WHAT
YOU NEED TO DO RATHER THAN
COMPLETELY REINVENT SOMETHING
NEW.
I WORRY A LITTLE BIT ABOUT THE
NIH SYNDROME, CALL IT NOT
INVENTED HERE SYNDROME IN PLAY
HERE.
SO I'M RAISING -- BEING GLASS
HALF EMPTY HERE BUT ON BEHALF OF
N CATS I'M CONCERNED HOW ARPA H
SETS ITSELF UP FOR SUCCESS, THE
GOAL IS LAUDABLE BUT THE -- IN
AN OPERATIONAL WAY TO GET THE
RIGHT PEOPLE YOU GOT TO PAY THEM
THE RIGHT AMOUNT TO GIVE THEM
THE AUTONOMY BUT IN THE
STRUCTURE OF THE NIH HOW ARE YOU
GOING TO MAKE THAT HAPPEN?
>> I THINK A LOT OF IT TIES BACK
TO THE SHORT ANSWER FOR ALL
THREE OF THOSE QUESTIONS, REALLY
TIES BACK TO AUTHORITIES THAT
ARE NEEDED.
THESE ARE THINGS WE HAVE BEEN
WORKING VERY CLOSELY WITH THE
HILL ON IN GIVING TECHNICAL
ASSISTANCE WHERE NEEDED.
TO PROVIDE RATIONALE FOR WHY WE
DO THINK THAT IT IS IMPORTANT
FOR ARPA H TO HAVE FOR EXAMPLE
REALLY ROBUST FLEXIBLE HIRING
AUTHORITIES.
BECAUSE AS YOU INDICATED, THERE
ARE LIMITATIONS THERE.
WE HOPE SOME LEVEL ONE OF THE
THINGS THAT WE WILL ARACKET
FOLKS TO IS THE ABILITY
ESPECIALLY WITH FIRST INSTRUCTOR
TO SHAPE AND MOLD THIS
ORGANIZATION BUT ALSO THE
POTENTIAL OPPORTUNITIES THAT
EXIST SO WE ARE GOING TO BE
CONFINED BY CERTAIN
RESTRICTIONS, OPERATING WITHIN
THE FEDERAL GOVERNMENT BUT
HAVING AS FLEXIBLE OF AUTHORITY
AS POSSIBLE WILL BE REALLY
HELPFUL.
SO HA IS THE FIRST POINT.
THE SECOND QUESTION, I'M FAILING
THIS COGNITIVE TEST, WAS AROUND
REALLY -- REMIND ME.
>> ABOUT THE ICs, WHERE WILL
IT STAND, IS THE STRUCTURE.
>> YEAH.
SO THE STRUCTURAL ORGANIZATION
THAT BEING CONSIDERED RIGHT NOW,
IT WILL HINGE ON THE LANGUAGE IN
THE FINAL BILLS FOR THIS BECAUSE
AGAIN THIS IS STILL PROPOSAL
PHASE, DOESN'T EXIST, IN DRAFT
FORM.
T IT IS BEING PROPOSED AS NOT
CALLING AN INSTITUTE, IT WON'T
BE LOCATED WITHIN THE OD.
I LIKE TO REFER TO IT AS ARPA H
BECAUSE IT IS UNIQUE.
IT IS DIFFERENT BUT IT WOULD BE
AT SIMILAR LEVEL AS AN IC.
I DON'T THINK WE HAVE TO WORRY
AT ALL KIND OF THE ISSUE OF
RESTRUCTURING NIH TO ACCOUNT FOR
THAT CAP, THAT IS SOMETHING THAT
WILL BE ADDRESSED AS NEEDED
WITHIN THE AUTHORIZING LANGUAGE
AND TO YOUR LAST POINT ABOUT THE
SYNERGIES AND COMMONALITIES OF
ARPA H COUPLE OF THINGS TO POINT
OUT, ARPA H DOES NOT INTEND TO
HAVE AN INTRAMURAL PROGRAM, SO
ONE THING THAT WILL MAKE IT
SOMEWHAT DISTINCT.
AMONG THE INSTITUTES AND
CENTERS.
BUT ALSO WHAT WE ARE TALKING
ABOUT HERE TOO IS LEVERAGING
DIFFERENT APPROACH AND MIND SET
TO FUNDING BIOMEDICAL RESEARCH,
THAT DRPA OR ARPA MODEL IF YOU
WILL AND WHILE I DO THINK THERE
ARE ELEMENTS THAT I THINK RING
TRUE AND PROBABLY SOUND SOMEWHAT
FAMILIAR FROM AN NCATS SIDE
THERE ARE DISTINCT DIFFERENCES
AND IT IS SOMETHING JONI AND I
TALK ABOUT HOW TO ENSURE THE
DISTINCTION AND THE COORDINATION
BETWEEN NCATS AND ARPA H THERE
AND AGAIN WE WANT THIS TO BE
COMPLIMENTARY SO WE ARE BUILDING
OFF THE DIFFERENT EFFORTS THAT
NOT JUST NCATS BUT ALL THE
ICs.
>> MAY I ASK A FOLLOW UP
QUESTION?
>> KELLY WILL HAVE THE LAST
WORD.
GO AHEAD.
>> I GUESS IF I HAIR YOU TALKING
ABOUT THAT HOW NCATS, MAYBE I'M
JUST TOO USED TO THINKING
STRUCTURE AND HOW THEY WORK.
JUST AS BARTA COULD BE PART OF
NIH BUT IT ISN'T.
GIVEN WHAT IT DOES.
IF YOU ARE GOING TO DO THIS WHY
NOT SET IT UP UNDER HHS AS
ANOTHER AGENCY EQUAL TO NIH
RATHERTHER HAHN SUBSUMING NIH,
NOT SURE WHAT -- YOU WANT A
DIFFERENT CULTURE, YOU WANT
SOMETHING DIFFERENT PRESUMABLY
NOT GOING TO PUT ON THE THE SAME
CAMPUS SAME BUILDINGS,
SUFFICIENTLY DIFFERENTLY SO WHY
SHORN IT?
>> SHOE HORN IT?
>> THREE REASONS, IT FITS WELL
WITH NIH.
THE MISSION OF ARPA H FITS WELL
AND SQUARELY WITHIN THE MISSION
OF NIH.
SO THAT'S ONE POINT.
THE SECOND POINT I WOULD SAY IS
TO LEVERAGE EXPERTISE AND
KNOWLEDGE THAT WE HAVE AT NIH
AND REALLY HELP TO FACILITATE
COLLABORATION.
THE FURTHER REMOVED YOU ARE, THE
HARDER TO BUILD THOSE.
WE HAVE SEEN INTERACTIONS WITH
BARTA.
TAKEN A LONG TIME TO GET TO
WHERE WE CURRENTLY ARE.
THE THIRD POINT IS MAYBE THE
MOST PRACTICAL.
THAT IS THAT WE -- STARTING THIS
WITHIN AN EXISTING ORGANIZATION
LIKE NIH ALLOW US TO LEVERAGE
INFRASTRUCTURE THAT EXISTS AND
WHAT I MEAN BY THAT, IS THE IT
NETWORKS, AGREEMENTS AROUND
GETTING SPACE AND REALLY JUST
PRACTICAL THINGS THAT ALLOW US
TO GET UP AND RUNNING IN A MUCH
QUICKER TIME FRAME THAN IF WE
HAD TO START COMPLETELY FROM
SCRATCH AS A NEW ORGANIZATION
WITHIN HHS.
IT WOULD SET US BACK YEARS.
>> I HAVE ONE OTHER QUESTION BUT
WILL LET KELLY GO FIRST.
>> WE COULD PROBABLY TALK ALL
DAY.
>> WE PROBABLY COULD.
IN EX TIME WE'LL HAVE MORE TIME
FOR YOU.
>> THANK YOU, TARA, THAT IS A
NICE PRESENTATION AND EVERYBODY
LIKES TO HEAR THE IDEA OF MORE
SPEED MORE URGENCY AND MORE
IMPORTANTLY MORE BUDGET TO FOCUS
ON BETTER HEALTH AND THE SIDE OF
THIS THAT IS ALSO ABOUT GLOBAL
COMPETITION FOR THE US. YOU
JUST STARTED GOING DOWN A PATH
AND I THOUGHT OH I CAN RETRACT
MY QUESTION I DON'T NEED TO ASK
BECAUSE YOU SAID THE PRACTICAL
REASON IS REALLY ABOUT SHARED
INFRASTRUCTURE.
I WONDER WHAT REDUNDANCIES WILL
BE MANAGED BY ARPA H WHEN YOU
START TO THINK THE BREAK THROUGH
INFRASTRUCTURE ALL THE WORDS
THAT YOU USE TALKING ABOUT THE
PLATFORM FOCUS PARTNERSHIPS AND
COLLABORATIONS, I REALLY WAITING
TO HEAR WHAT IS GOING TO BE
REALLY DIFFERENT I FEEL LIKE
NCATS DOES ALL OF THAT AND I
THINK THE BREAK THROUGH
INFRASTRUCTURE THEY HAVE IN
PLACE AND I DON'T THINK THAT
THAT IS A TERM USED ANYWHERE BUT
THAT IS SORT OF WHAT HAS BEEN
SET UP OVER THE PAST DECADE,
WILL THAT BE LEVERAGED, THERE
WILL BE RECONDITION DANCIES IF
IT IS GOING TO NOT INCLUDE
INTRAMURAL RESEARCH WILL
EXTERNAL PEOPLE WORK AND
COLLABORATE TO HAVE ACCESS TO
SOME OF THESE RESOURCES THIS
INFRASTRUCTURE THAT DOESN'T
EXIST ANY PLACE ELSE IN THE
WORLD EXCEPT BIG PHARMA.
COULD YOU COMMENT ON THAT A
LITTLE BIT?
THAT GETS INTO SOME OF THE
PRACTICAL BRASS TAX THAT HAS NOT
BECOME CLEAR TO ME YET.
>> I THINK EVERYTHING YOU JUST
LAID OUT IS ABSOLUTELY CORRECT.
THE LEVERAGING THE
INFRASTRUCTURE THAT EXISTS HERE
IS GOING TO BE A KEY ELEMENT OF
ARPA H.
I THINK THAT WE WOULD EXPAND
THAT EVEN FURTHER TO TALK ABOUT
SOME OF THE INFRASTRUCTURE THAT
EXISTS ACROSS THE FEDERAL
GOVERNMENT BECAUSE I THINK THERE
IS A WHOLE LOT MORE WE COULD DO
IN OPERATING IN SORT OF MUCH
BETTER COORDINATION AS A WHOLE.
EXACTLY WHAT YOU SAID, THE IDEA
IS IS THAT WE WOULDN'T BE
SETTING UP OUR OWN CLINICAL
TRIAL NETWORKS OR OTHER THINGS
THAT ALREADY EXIST WITHIN NIH
BUT RATHER I VIEW IT AS A
FEEDBACK LOOP.
MAYBE EXAMPLE THIS IS NOT
LIMITING BUT SOME OF THE REALLY
INTERESTING AND EXCITING
FUNDAMENTAL BASIC RESEARCH
THAT'S COMING OUT OF THE
INSTITUTES FEEDS INTO OR ALLOWS
FOR THE IDEA ARPA H HAS TO MOVE
FORWARD AND MAKE STRIDES AND
ADVANCEMENTS AND THEN MAYBE YOU
TAKE THAT AND GET THAT TO PROOF
OF CONCEPT STAGE, AND YOU
LEVERAGE AN ICs CLINICAL TRIAL
NETWORK AND WORK WITH FDA AND
CMS AND OTHERS ACROSS THE
FEDERAL GOVERNMENT TO MOVE THAT
FORWARD IN A ROBUST WAY.
THERE MIGHT BE THEN AN
OPPORTUNITY AT THE END TO
OBVIOUSLY THEN HAVE ANOTHER
MAYBE EVEN ARPA H PROJECT WHERE
WHAT DOES THAT LOOK LIKE TO
ADDRESS SOME MAYBE LARGE SCALE
NATIONAL CHALLENGES TOWARDS
IMPLEMENTATION.
I THINK THE OTHER THING TO POINT
OUT TOO IS THAT WITH ARPA H WHAT
WE ARE TALKING ABOUT IS
BIOMEDICAL AND HEALTH RESEARCH,
WE USE THAT TERMINOLOGY REALLY
BROADLY BECAUSE WE SORT OF VIEW
IT FROM REALLY LARGE SPECTRUM OF
THE TYPE OF RESEARCH THIS WILL
SUPPORT AND GET INTO
IMPLEMENTATION AND DISSEMINATION
AND HOW THESE THINGS GET OUT TO
PATIENTS.
>> THANK YOU.
>> LAST QUESTION OR IMMEDIATE TO
CLOSE OUT?
>> WE NEED TO CLOSE OUT.
GO AHEAD, ASK YOUR QUESTION
PLEASE.
>> I WANT TO COME TO MONEY.
SO YOU ARE MAKING THIS ASK IN
THE BUDGET WHATEVER THE NUMBERS
YOU SAID WHETHER IT IS WE HEARD
DIFFERENT NUMBERS THROWN AROUND,
3 BILLION, 6 BILLION WHATEVER
THAT MIGHT BE.
HARKENING BACK TO EXPERIENCE
STARTING N CATS AND WHAT WE GOT
INITIALLY AND HOW WE GET IT TO
THE POINT AND ONE OF THE THINGS
THAT WAS A THIRD RAIL IN THE
ORGANIZATION WHEN NCATS WAS
STOOD UP IS THE IDEA THAT HE PUT
IN ANY WAY CANNIBALIZE ANY OTHER
IC BUDGET SO IN A SENSE THAT
THERE IS A ZERO SUM CONCERN THAT
THIS HAD TO BE NEW MONEY HA
COMES IN TO DO THIS.
SO ARE WE SAYING THAT THAT IS
WHAT REALLY IS GOING TO HAPPEN
WITH THE POLITICAL CLIMATE
CERTAINLY SUPPORT ONE FIFTH OF
THE NIH BUDGET TO BE ARPA H,
THAT'S THE SIZE OF ESSENTIALLY
THE LARGEST INSTITUTE LIKE NCI
AND NIAID WOULD BE STOOD UP FROM
THE GET GO.
>> I MEAN IF YOU -- LOOK AT THE
DRAFT LEGISLATION, IT IS ALL
DRAFT LANGUAGE IN TERMS OF
APPROPRIATION.
IT IS NEW MONEY, IT IS NOT
CANNIBALIZING FROM THE NIH
BUDGET.
WE WOULD NOT ADVOCATE FOR THAT
IN FACT WE DIDN'T, WE NAVIGATED
HARD THAT THAT SHOULDN'T BE THE
CASE.
IF FACT I THINK BOTH THE HOUSE
AND SENATE GAVE THE NIH
INCREASES ON TOP OF THE FY 21
BUDGET FOR FY 22.
THIS IS ALL DRAFT LANGUAGE,
NOTHING HAS BEEN PASSED YET.
>> THANKS, THAT WAS TERRIFIC.
YOU EXPERTLY ANSWERED A LOT OF
QUESTIONS AND WE REALLY
APPRECIATE YOUR TIME HERE.
THANK YOU FOR THE PRESENTATION.
WE LOOK FORWARD TO SAYING ALONG
WITH YOU HOW WE UPHOLD THIS
GOING FORWARD AND HOPEFULLY WE
CAN HAVE THIS BACK SOON AT
COUNCIL.
>> I APPRECIATE COPY OF THAT
REPORT MENTIONED AT THE
BEGINNING, WOULD LOVE TO SEE
THAT.
THANK YOU FOR INVITING ME.
APPRECIATE THE ROBUST DISCUSSION
AND I'M ASSURE JONI YOU AND I
WILL TALK AGAIN.
TAKE CARE.
>> THANK YOU.
>> THANK YOU SO MUCH.
WITH THAT WE DID GET A LITTLE
OVER IN TIME SO HOPE WE CAN MAKE
UP FOR THAT IN THE NEXT COUPLE
OF PRESENTATIONS.
BUT WE ARE GOING TO MOVE ON TO
LILY PORTILLA I BELIEVE IS NEXT
TO TALK ABOUT THE OFFICE OF
STRATEGIC ALLIANCE.
SO LILY PLEASE GO AHEAD.
>> CAN YOU HEAR ME?
>> YEP.
>> GREAT.
HI, I'M LILY PORTILLA, DIRECTOR
OF STRATEGIC ALLIANCES AT NCATS
AND JUST TO ORIENT TO COUNCIL
OUR OFFICE IS IN CHARGE OF ALL
THE AGREEMENT AND PARTNERSHIP
ESTABLISHMENTS THAT GO ON ACROSS
THE CENTER, THE OTHER THING WE
DO IS ALSO RUN THE SBIR STTR
PROGRAM AND THE FOCUS OF MY
CONVERSATION TODAY OR MY
PRESENTATION TODAY IS GOING TO
BE ON THE SBR STTR PROGRAM AND
OUR EFFORTS AROUND THAT SPECIFIC
PROGRAM, WITH THAT I WILL START.
NEXT SLIDE PLEASE.
SO THE NIH SBIR STTR PROGRAMS
LARGEST SOURCES OF EARLY STAGE
SEED FUNDING IN THE COUNTRY.
BUDGET IS WELL OVER A BILLION
DOLLARS NOW THAT ACROSS THE NIH
THAT HAS TO GO TO U.S. SMALL
BUSINESSES WORKING IN THE
BIOMEDICAL SPACE AND 24
INSTITUTES INCLUDING NCATS HAVE
BUDGETS THAT SUPPORT THE SBIR
STTR PROGRAM.
IT IS A 3.65% SET ASIDE, IT IS A
CONGRESSIONALLY MANDATED
PROGRAM, IT IS NOT MONEY THAT
CAN BE REPROGRAMMED TO OTHER
GRANT LINES OR CONTRACT LINES,
IT HAS TO BE USED IN SUPPORT OF
U.S. SMALL BUSINESS.
LET'S GO TO NEXT SLIDE.
LET'S CLICK ONE MORE TIME.
MAJORITY OF APPLICATIONS THREE
WAYS APPLICATIONS COME IN FOR
THIS PROGRAM.
THE MAJORITY OF THE APPLICATIONS
COME IN WHAT'S CALLED THE
OMNIBUS THAT IS 24 PARTICIPATING
INSTITUTES UTILIZES WELL AS OUR
SISTER AGENCIES AT THE CDC AND
FDA, OTHER PARTS OF HHS.
I WOULD SAY ABOUT 90%
APPLICATIONS THAT WE GET IN ARE
FUNDED THROUGH THE OMNIBUS WHICH
IS BROAD TOPICS ACROSS THE NIH
NCATS HAS LISTED THOSE RESEARCH
PRIORITIES AS PART OF THE
OMNIBUS SOLICITATION.
THE OTHER WAY WE GET
APPLICATIONS IS LIEU TARGETED
SOLICITATION, WE HAVE A FEW OF
THEM WE GENERATED HERE AT N
CATS, WE ALSO PARTICIPATE IN
SOLICITATION OTHER INSTITUTES
HAVE PUT TOGETHER.
ONCE A YEAR THERE IS A CONTRACT
SOLICITATION AND THIS IS WHEN
SPECIFIC DELIVERABLE -- WE
BELIEVE A SPECIFIC DELIVERABLE
CAN BE TARGETED BY SMALL
BUSINESS AND I WANT TO CLARIFY
THAT IT IS NOT LIKE A SERVICE,
WE ARE NOT ASKING FOR A SERVICE
TO BE PROVIDED BY A SMALL
BUSINESS BUT WE ARE SAYING THAT
WE THINK THERE'S A TECHNOLOGY
SOLUTION PROVIDED BY SMALL
BUSINESS AND THE CONTRACT
MECHANISM WHICH HAPPENS ONCE A
YEAR, IS BEST WAY THE GET THOSE,
WE BROAD THOSE TO YOU ALL AND
COUNCIL THE APPROVE.
SBIR STTR HAS SEVERAL BENEFITS
AS I MENTIONED LEAD TO OVERALL
BUDGET SO IT IS STABLE AND
PREDICTABLE,IT IS NON-DILUTIVE.
THE GOVERNMENT DOESN'T TAKE IP
RIGHTS DEVELOPED UNDER THIS
PROGRAM.
ONCE IN THE PROGRAM, ONCE YOU
RECEIVE A GRANT YOU GET ACCESS
TO SOME TECHNICAL ASSISTANCE AND
COMMERCIALIZATION PROGRAMS THAT
THE NIH OFFERS ALONGSIDE OF WITH
THE GRANT WE FOR EXAMPLE OFFER
COMPANIES OPPORTUNITY TO GO
THROUGH INNOVATION CORE TRAINING
PROGRAM.
WE WILL PAY FOR THEM TO DO THAT.
WE ALSO NIH HOST IN LARGE
CONFERENCES LIKE BIO AND ABMED
HAVE SPACE WHERE MANY COMPANIES
CAN DO PITCHES TO POTENTIAL
INVESTORS AS WELL TOO, THAT IS
ANOTHER BENEFIT OF BEING IN THE
PROGRAM.
I BELIEVE I MENTIONED THAT
BEFORE THAT, ONE OF THE OTHER
BENEFITS IS THAT THE PROGRAM --
THE GRANTS AND CONTRACTS GO
THROUGH RIGOROUS PEER REVIEW
PROCESS, WHICH THEY CAN THEN
LEVERAGE FOR ADDITIONAL FUNDING
THROUGH OTHER SOURCES, PRIVATE
INVESTMENT, AND EVEN STATE
ECONOMIC ORGANIZATIONS HAVE
COMPLIMENTARY PROGRAMS ONCE YOU
GET AT NIH SBIR STTR.
IT IS THREE PHASE PROGRAM, PHASE
1 IS FEASIBILITY STUDIES AND
THERE ARE PROGRAM CAPS BUDGET
CAPS THAT THE NIH HAS TO ADHERE
TO THAT ARE SET BY THE SMALL
BUSINESS ADMINISTRATION.
HOWEVER, THERE ARE SOME TOPICS
ACROSS THE NIH THAT WE HAVE
ALLOWED HIGHER BUDGET DOLLAR
AMOUNTS, PHASE 2 IS MORE FULL
ADD PROGRAMS WE CAN GO UP TO 2
MILLION OVER PROJECTS THAT LAST
BETWEEN TWO TO THREE YEARS.
THE FAST TRACK THAT COMBINES
BOTH THE PHASE 1 AND PHASE 2 ONE
REVIEW THE COMPANY HAS TO GO
THROUGH.
DIRECT TO PHASE 2 WHICH ALLOWS
YOU TO SKIP THE PHASE 1 IF YOU
HAVE ENOUGH DATA YOU HAVE GOTTEN
UNDER FEASIBILITY STUDY.
AND THE PHASE 2B COMPETING
RENEWAL PROGRAM WE OFFER TO ONLY
COMPANIES THAT WE FUND IN PHASE
2.
UNLIKE OTHER AGENCIES WITH STIR
SBIR, WE DO NOT HAVE A PHASE 3
PROGRAM BUT WE PRESUME COMPANIES
ARE GRADUATING OUT OF THE
PROGRAM AND PSYCHING PRIVATE
SOURCES OF FUNDING, OR
DEVELOPING STRATEGIC
PARTNERSHIPS.
HOPEFULLY THE GRANT HAS GOT TO
THAT KEY INFLECTION POINT WHERE
THEY CAN DO THAT.
NEXT SLIDE.
THERE ARE -- I WON'T GO THROUGH
THE DIFFERENCES BETWEEN
GUIDELINE AND ELIGIBILITY
REQUIREMENTS BUT THERE ARE --
THE MAIN DIFFERENCE BETWEEN THE
TWO OF SBIR AND STTR IS THE STTR
HAS -- MUST HAVE A RESEARCH
INSTITUTION COMPONENT TO IT.
IN THAT INSTANCE THE PI CAN
EITHER WORK FOR THE RESEARCH
INSTITUTION OR CAN WORK FOR THE
SMALL BUSINESS.
BUT KEEP IN MIND THAT THE GRANT
ALWAYS HAS TO GO TO THE SMALL
BUSINESS.
THERE ARE WORK REQUIREMENTS
DIFFERENCE BETWEEN PROGRAMS NOT
ALL GRANT CAN BE OUTSOURCED,
MANY TIMES WE HAVE VIRTUAL
COMPANY ASKING IF THEY WANT TO
PARTICIPATE IN THE PROGRAM BUT
THERE HAS TO BE WORK DONE WITHIN
THAT SMALL BUSINESS IN ORDER TO
BE ELIGIBLE TO RECEIVE THE
AWARD.
NOW I WILL GET INTO INITIATIVES
AND PROGRAM CHANGES ACROSS THE
NIH.
NEXT SLIDE.
THERE HAVE BEEN CHANGES TO THE
BUDGET CAPS, THERE WAS A PERIOD
OF TIME THERE WHERE THE BUDGET
CAP STAYED STATIC OF SMALL
BUSINESS ADMINISTRATION OVER
SEVERAL YEARS HAS INCREASED THAT
WE ARE NOW UP TO 275 AND CHANGE
FOR PHASE 1 AND 1.8 MILLION FOR
PHASE 2.
THERE ARE TOPICS AT THE NIH DOES
ALLOW FOR HIGHER BUDGET AMOUNTS
AND THEY ARE CALLED WAIVER
TOPICS.
EACH INSTITUTE HAS THEIR WAIVER
TOPICS THAT YOU CAN APPLY UNDER
AND FOR US THE WAIVER TOPICS
ALLOW YOU TO GO UP TO 325,000
FOR PHASE 1.
2 MILLION FOR PHASE 2.
WE THINK THAT THAT IS WARRANTED
FOR SOME TOPICS BUT WE BELIEVE
OUR BUDGET CAN SUPPORT THESE
HIGHER BUDGET DOLLAR AMOUNTS.
NEXT SLIDE PLEASE.
THERE'S ALSO CHANGES AROUND
TECHNICAL AND BUSINESS
ASSISTANCE PROGRAM.
COMPANIES NOW REQUEST IN
ADDITION TO THE -- THEIR BUDGET
THEY CAN ASK FOR 6,500 PER YEAR
PHASE 1 AND 50,000 FOR PHASE 2,
IT WOULD ALLOW THE COMPANY TO DO
THINGS LIKE MARKET ANALYSIS, OF
TECHNOLOGY, PATENT LANDSCAPE OR
FILING PATENTS BUT THIS IS A NEW
INITIATIVE THAT IS STARTED NIH
OFFERS COMPANIES THAT ARE
INTERESTED CONSULTING SERVICES
TO FIGURE TECHNOLOGY TO A
INFLECTION POINT TO GET
STRATEGIC PARTNERSHIP OR
INVESTMENT BUT THE TECHNICAL
ASSISTANCE FUNDING IS VERY
HELPFUL IN TERMS OF DOING THAT.
NEXT SLIDE PLEASE.
I ALSO WANT TO POINT OUT THAT
LIKE OTHER NIH GRANT PROGRAMS
SBIR STTR TAKES ADMINISTRATIVE
SUPPLEMENTS TO PROMOTE
DIVERSITY.
IN THIS INSTANCE THE -- THIS
PARTICULAR ADMINISTRATIVE
SUPPLEMENT IS IN SUPPORT OF
INDIVIDUALS WHO WANT TO WORK IN
SMALL BUSINESS AND GET
EXPERIENCE AS PART OF THE C
SUITE OR DOING RESEARCH FOR THE
SMALL BUSINESS AND I WANT TO
NOTE THIS IS A SPECIAL
ADMINISTRATIVE SUPPLEMENT ONLY
FOR SBIR STTR COMPANIES.
WE ALSO PARTICIPATE WITH NINE
OTHER INSTITUTES IN THE
APPLICANT ASSISTANCE PROGRAM AND
HERE APPLICANTS THAT HAVE EITHER
NEVER APPLIED TO THE NIH OR
APPLIED NOT SUCCESSFUL AND GIVEN
A MENTOR TO WORK WITH PUTTING
APPLICATION TOGETHER, HONING
TOPICS AND THE CONSULTANT THAT
WORKS WITH THEM HAS A LOT OF
EXPERIENCE WORKING WITH --
HELPING COMPANIES WITH NIH
GRANTS AND WHAT IT DOESN'T DO,
IT DOESN'T WRITE THE GRANT FOR
THEM BUT GIVES THEM THE TOOLS
AND KNOWLEDGE NEEDED TO BE
SUCCESSFUL IN GETTING NIH
FUNDING.
THE PROGRAM DOES HAVE A FOCUS ON
SUPPORTING WOMEN AND MINORITY
OWNED SMALL BUSINESSES AS WELL.
TO THE NEXT SLIDE.
HERE WE KIND OF TALK I THINK I
MENTIONED A FEW THINGS HERE,
THAT IF YOU ARE TO BE ELIGIBLE
YOU HAVE NEVER GOTTEN NIH GRANT,
COMPANY DOESN'T HAVE CURRENT
APPLICATION UNDER REVIEW, WE
AGAIN TRY TO MAKE SURE THAT
THESE INTERESTS OF FOCUS OF THE
APPLICATIONS ALIGN WITH THOSE
INTEREST OF THE PARTICIPATING
INSTITUTES.
MENT WE ARE LOOKING AT THE
PROGRAM OVERALL TO DETERMINE
WHETHER IT IS SUCCESSFUL AND
HOPEFULLY NEXT TIME WE MEET I
WILL BE ABLE TO PROVIDE MORE
DATA AROUND THAT.
WE FOLLOW SOMETHING LIKE REALLY
WOULD BE A GOOD INVESTMENT OF
TIME AND ENERGY TO TRY TO FIGURE
OUT HOW TO HELP CERTAIN
APPLICANTS GET OVER THIS HUMP OF
GITTING FUNDING FOR UNDER THIS
SPECIFIC PROGRAM.
SO INITIATIVES WE ARE DOING
AROUND OUTREACH AND DRIVING
AWARENESS TO THE NCATS SBIR STTR
PROGRAM, NEXT SLIDE SO OUR
OBJECTIVES ARE GETTING PEOPLE TO
BE AWARE OF WHAT TYPES OF
PROJECTS WE ARE FUNDING UNDER
THE PROGRAM, TO INCREASE THE
NUMBER OF APPLICATIONS THAT
WOMEN OWN AND MINORITY OWNED
SMALL BUSINESSES APPLY TO US
INCREASING THOSE NUMBERS OF
QUALITY APPLICATIONS, ALSO SMALL
BUSINESSES IN STATES THAT
TYPICALLY DON'T GET A LOT OF NIH
FUNDING EITHER TRYING TO FOCUS
EFFORTS AND OUTREACH AROUND THAT
TOO, IN GENERAL ADVANCING
INVASION AND DRUG DEVELOPMENT
DISCOVERY RESEARCH TOOLS AND
TECHNOLOGIES THAT IMPROVE
PATIENT CARE AND THOSE RESEARCH
TOPICS THAT ARE OF GREAT
IMPORTANCE AND PRIORITY TO
NCATS. NEXT SLIDE.
THIS PAST YEAR EVEN THOUGH WE
WEREN'T ABLE TO DO IN PERSON
OUTREACH WE DID A FAIR AMOUNT OF
WEBINARS WE WERE QUITE BUSY AND
PARTNERED WITH VARIOUS GROUPS TO
GET WEBINARS IN PLACE, STATE BIO
ORGANIZATIONS, AS WELL AS
ACADEMIC TECH TRANSFER OFFICES,
INNOVATION HUBS AND ACCELERATORS
AND GOVERNMENTAL PARTNERS WE
WORKED WITH TO DO JOINT OUTREACH
EVENTS.
I WANT TO POINT OUT WE HAVE BEEN
BUSY THOUGH NOT ABLE TO DO IN
PERSON OUTREACH.
THIS PAST YEAR WE WERE ABLE TO
GET PARTICIPATION TO WEBINARS
FROM 45 STATES, AND 45 SATES
WERE IDEA STATES, WHICH WE WERE
PURPOSEFULLY TARGETING.
THIS PAST YEAR WE TALKED TO A
HUNDRED APPLICANTS AND -- I WILL
TALK ABOUT HOW WE TRACK WHAT
HAPPENS AFTER THOSE
CONVERSATIONS WHAT HAPPENS TO
THE APPLICANTS BUT THIS YEAR WE
WANT TO TARGET OTHER STATES WE
WEREN'T ABLE TO MAKE IN ROADS IN
AND THERE'S ONLY A FEW OF THEM
HERE THAT WE HAVE LISTED.
BUT THAT IS PRIORITY NUMBER ONE
GOING INTO FY 22.
NEXT SLIDE.
WE HAVE A SOCIAL ENGAGEMENT
CALENDAR WE PUT TOGETHER EVERY
MONTH WITH OUR COMS TEAM.
WE HAVE GOTTEN TRAFFIC AS A
RESULT OF THE EFFORTS WE HAVE
BEEN DOING EVERY TUESDAY WE HAVE
A TIP TUESDAY SOCIAL ENGAGEMENT
THAT GOES OUT ABOUT HOW TO PUT
IN AN APPLICATION TOGETHER,
ADVISING APPLICANTS WHAT TO DO
FOR PARTICULAR FUNDING
ANNOUNCEMENT.
WE HIGHLIGHT NCATS STAFF
INVOLVED WITH THE PROGRAM
UPCOMING WEBINARS.
ANOTHER THING THAT IS VERY
SUCCESSFUL ARE THESE QUOTE CARDS
HIGHLIGHT SOME OF OUR GRANTEES
AND SPECIFICALLY SOME HIGHLIGHT
AROUND COMPANIES, MILESTONE THEY
REACHED IF WE CAN GO TO THE NEXT
SLIDE.
BY WAY OF EXAMPLE, THIS PAST
YEAR WE HAVE WORKED CLOSELY WITH
OUR PROGRAM OFFICERS AND ALSO
HAVE ALERTS SO WE KNOW WHEN
COMPANIES WE HAVE FUNDED HAVE
GOTTEN ACANNINGAL FUNDING OR
CREATED A STRATEGIC PARTNERSHIP,
WE TALKED TO THAT GRANTEE, TO
FIND OUT WHAT IS GOING ON.
AND AMPLIFY THE FACT THAT THEY
HAVE REACHED A MILESTONE.
GREAT WAY ENGAGING COMPANY BUT
ALSO OTHERS ABOUT GOOD WORK WE
ARE DOING WITH PROGRAM AND
ENTREPRENEURS THAT WE ARE
FOCUSING AS PART OF THE SOCIAL
ENGAGEMENT OPPORTUNITIES HERE.
NEXT SLIDE.
/TALK ABOUT FACT WE HAVE HAD --
WE DID 100 INDIVIDUALCALS WITH
INDIVIDUALS WHO NEEDED TO TALK
TO US ABOUT PROJECTS AND WE --
WHEN WE FIRST STARTED OUT WE HAD
A HARD TIME TRACKING WHO WE
SPOKE TO BUT MORE IMPORTANTLY
WHAT HAPPENED AFTER THAT
CONVERSATION.
SO NOW WE ARE UTILIZING SALES
FORCE TO GIVE US AN IDEA OF WHEN
THESE CONVERSATIONS HAPPEN, HOW
LONG DOES IT TAKE THE APPLICANT
TO I APPLY TO THE PROGRAM AND
ONCE THEY APPLY, HOW SUCCESSFUL
WERE THEY.
ARE THEY APPLYING FOR OTHER
GRANTS AND NOW THIS -- OUR SALES
FORCE APPLICATION IS ABLE TO
TALK TO THE NIH SYSTEM SO WE ARE
ABLE TO SEE REAL TIME GRANTS AT
THESE POTENTIAL APPLICANTS HAVE
AND TRACK WHAT CONVERSATIONS
HAVE LED TO.
IT IS A GREAT TOOL TO KEEP TRACK
OF CERTAIN CAMPAIGNS THAT WE ARE
DOING WEBINARS, WHAT'S BEEN THE
INTEREST AROUND PARTICIPATION
POST WEBINAR WITH FOLKS COMING
TO NCATS, APPLYING TO THE
PROGRAM.
I WILL GIVE DATA, I TALKED
INITIATIVES AROUND THE PROGRAM
SO NOW THIS TIME THE TALK TO
DATA SO LET'S DO THAT.
QUICKLY TO GIVE YOU SENSE OF
PROGRAM, IT IS 25 MILLION THAT
GOES TO FUNDING SMALL BUSINESSES
SBIR STTR.
AT ANY GIVEN YEAR E WE HAVE
ABOUT I WOULD SAY LIKE A 15 TO
80 -- 15 OR 20% OF OUR BUDGET
GOES TO FUNDING THOSE SBIR
CONTRACTS THAT I MENTIONED
EARLIER AND THE REST REALLY GOES
TOWARDS SUPPORTING THE GRANT
PROGRAM.
TO THE NEXT SLIDE.
IF YOU WERE TO LOOK AT OUR
PORTFOLIO THIS PACIFIST WALL
YEAR I WOULD SAY THAT THEY FALL
INTO FOUR BUCKETS THE MAJORITY
ARE AROUND TOOLS AND
TECHNOLOGIES FOR DRUG DISCOVERY
AND DEVELOPMENT, IT IS A BROAD
TOPIC, WE ALSO HAVE DEVICE
PORTION OF OUR PORTFOLIO A LOT
OF OUR TISSUE CHIP FALLS UNDER
THAT SLIVER OF DEVICE.
GENE THERAPY BECAUSE WE HAVE A
FOCUS PROGRAM ANNOUNCEMENT
AROUND GENE THERAPY AND THEN THE
REST AROUND BIOINFORMATICS.
SO THIS IS SHOWING OVER TIME THE
NUMBERS OF APPLICATIONS THAT WE
HAVE BEEN GETTING IN AND COUPLE
OF OBSERVATIONS.
ONE WE DON'T TALK ABOUT THIS
PROGRAM WE DON'T SEE -- PEOPLE
DON'T APPLY SO YOU HAVE TO IN
ORDER TO GET PEOPLE/NEW FRESH
GRANTEES TO THE MIX YOU NEED TO
BE TALKING ABOUT THE PROGRAM AND
DOING OUTREACH ON A PRETTY
CONSISTENT BASIS.
THE OTHER THING WE NOTICED WAS A
DROP IN 2020 OF APPLICATIONS AND
MANY APPLICANTS THOUGH HOME
BOUND HAD TIME, THEY HAD OTHER
RESPONSIBILITIES, I KNOW THAT
ANECDOTALLY SEVERAL APPLICANTS
SAID IT WAS HARD TO WRITE A
GRANT WHEN THEY WERE HAVING TO
TAKE CARE OF KIDS OR PARENTS
DURING COVID.
SO WE DID SEE A DROP IN
APPLICATIONS HAPPENING AND ALSO
SOME OF OUR COMPANIES WERE
IMPACTED BY THE FACT THAT THEY
COULDN'T HIRE DURING THE
PANDEMIC SO THAT DELAYED THEM
PERHAPS MOVING FROM THE PHASE 1
TO THE PHASE 2.
IN 2021 BESTARTED TO SEE AN
INCREASE BACK -- WE STARTED TO
SEE AN INCREASE BACK AGAIN IN
NUMBER OF APPLICATIONS THAT CAME
TO US.
NEXT SLIDE.
HERE WE ARE FOCUSING ON AWARDED
APPLICATIONS.
TYPICALLY AT ANY GIVEN TIME I
THINK WE RUN ANYWHERE BETWEEN 25
TO 35 APPLICATIONS THAT WE --
NEW APPLICATIONS WE FUND EVERY
YEAR.
NEXT SLIDE.
HOW DO -- WHAT ARE -- DOES OUR
PRIORITY SCORE, WHAT ARE WE
FUNDING THE QUESTION WE GET
OFTEN AND I THINK OVER TIME WE
HAVE SEEN IMPROVEMENT IN THE
AVERAGE PRIORITY SCORES NCATS
FUNDS IN SBIR, AGAIN PRETTY
PREDICTABLE WHAT OUR BUDGET IS
GOING TO BE SO WE KNOW WHETHER
KIND OF OUTREACH THAT WE HAVE TO
DO IN OUR APPLICANTS -- WE HAVE
REPEAT GRANTEES WHO COME BACK TO
US AND FUND VARIOUS PROJECTS.
SO I DO THINK THAT IT IS
LEVELING OFF AND THIS IS KIND OF
WHAT FY 20 -- FY 21 WAS VERY
COMPETITIVE YEAR.
BECAUSE WE HAVE SEEN OUR
APPLICANTS NOT COME IN FOR
ADDITIONAL FUNDING BECAUSE THEY
HAVEN'T BEEN ABLE TO FINISH
THEIR AIMS AND GRANT BECAUSE OF
COVID.
I DO EXPECT IT TO BE A DIFFERENT
NUMBER THIS NEXT IN THISY 2022.
WE WILL SEE WHAT HAPPENS SO WHAT
DO WE LOOK LIKE COMPARED TO THE
REST OF NIH?
NEXT SLIDE.
WE ARE CLOSE.
SOME YEARS WE ARE DOING BETTER
THAN WHAT NIH AVERAGE PRIORITY
SCORES ARE FOR SBIR AND SOME
YEARS WE ARE DOING A LITTLE BIT
WORSE.
CONSISTENTLY WE ARE STAYING
ALONG THE LINES OF WHAT NIH
FUNDS FOR SBIR.
TO THE NEXT SLIDE.
EYE A LITTLE BIT DIFFERENT.
COUPLE OF REASONS.
ONE IS THAT OUR BUDGET IS NOT
THAT HIGH FOR STTR SO OUR
APPLICANTS COME IN AND FIGURE
AND SAY I WILL TAKE A CHANCE AND
GOING TO APPLY FOR SBIR BECAUSE
I FEEL LIKE I HAVE A BETTER
CHANCE THERE.
SOME YEARS WHAT WE SEE IS DROP
OFF IN NUMBER OF APPLICATIONS
THAT COME IN UNDER SPECIFIC --
UNDER STTR.
OTHER YEARS WE SEE A FEW PHASE
2s TAKE UM THE BUDGET WHICH
LIMITS WHAT WE CAN FUND. SO
FREE YEAR-TO-YEAR THERE IS
FLUCTUATION WITH WHAT WE FUND
UNDER THE PROGRAM.
SHOWING HOW WE ARE DOING WITH
REST OF NIH, I THINK IT HAS BEEN
A LITTLE ALL OVER PLACE.
DEPENDS ON WHETHER THERE'S A
STREAM OF APPLICATIONS COMING
IN, WHETHER THERE'S PHASE 2s
TO FUND.
APPLICANTS COMING IN SAYING I'M
GOING TO PUT TOGETHER ABIR
VERSUS STTR.
ANOTHER THING WE HAVE BEEN
TRACKING TO IS WOMEN OWNED AND
MINORITY OWNED SMALL BUSINESSES
COMING AND APPLYING.
DROVES A DROP OFF IN 2019.
WE ACCOUNT THAT TO CHANGE IN HOW
DATA IS COLLECTED.
THE DATA USED TO BE COLLECTED AT
THE NIH LEVEL WITHIN YOU PUT
YOUR APPLICATION IN, YOU WOULD
TAKE WHETHER YOU WERE MINORITY
OWNED OR WOMAN OWNED SMALL
BUSINESS VERY DEPENDENT ON SBA
GUIDELINES.
NOW THAT DATA IS CAPTURED ON THE
SBA SITE SIDE WHEN YOU REGISTER,
YOU DO YOUR SBA REGISTRATION.
AND WE HAVE SEEN A SHIFT IN THE
NUMBERS.
IF YOU LOOK LOOK AT HER PARTS OF
NIH YOU SEE SIMILAR SHIFTS AS
WELL TOO IN TERMS OF DATE
CAPTURE.
TO THE NEXT SLIDE.
I WILL END WITH SUCCESSORRIES.
AS PART OF THE TEN YEAR
ANNIVERSARY WE TALKED ABOUT WHAT
WE DID ON I BELIEVE DECEMBER 7
WE HAD THIS BIG LOT OF
PARTICIPATION AROUND THE TEN
YEAR ANNIVERSARY BUT ALSO
DECIDED TO DO SOMETHING WITH
SBIR TOO AND HIGHLIGHT THREE
SUCCESS STORIES WITH COUPLE OF
OF OUR GRANTEES.
LET ME GO THROUGH SOME OF THE
STORIES.
I WANT O GET A GOOD
REPRESENTATION OF DIFFERENT
PARTS OF THE ENTREPRENEURIAL
CYCLE TO GIVE ILLUSTRATE HOW
THESE COMPANIES USE THE GRANT
MORE IMPORTANTLY HOW THEY HAVE
GOT TO KEY INFLECTION POINTS
ALONG THE WAY -- MOVING THEIR
TECHNOLOGY FOR
COMMERCIALIZATION.
LET'S GO TO NEXT SLIDE.
WE HAD DR. LIN PARKS WU WHO
DEVELOPED THIS PORTABLE SYSTEM
CALLED THE BLAZE SYSTEM WHICH
COMBINES MASS SPEC WITH PROTEIN
IMAGING AND LEONA WAS ABLE TO DO
THIS WITH DEVELOPMENT WITH SBIR
BUT SHE SOLD HER COMPANY TO
(INAUDIBLE) AS A RESULT OF THAT
SO THE COMPANY WAS INTEREST
MISDEMEANOR THE PRODUCT AND HER
EXIT STRATEGY WAS TO SELL THE
ASSET WHICH IS WHAT SHE DID SHE
WAS SUCCESSFUL IN DOING THAT BUT
SBIR HELPED WITH THE EARLY
DEVELOPMENT OF THE TECHNOLOGY.
THE NEXT PERSON WAS CHRIS
GIBSON, CHRIS SURE YOU HAVE
HEARD ABOUT RE,CURSION.
ONE OF THE FIRST COMPANIES WE
FUNDED WHEN WE STARTED THE
CENTER.
IT EAT A GREAT STORY BECAUSE
THAT INITIAL GRANT THAT CHRIS
WENT FOR OVER TIME COMPANY WAS
ABLE TO LEVERAGE INVESTMENT,
CHRIS'S COMPANY WAS SUPPORTED BY
NIH GOING TO VARIOUS PITCH
PETITION THAT BIO HAD AND OTHER
CONFERENCES HE COULD SHOWCASE
WHAT HE WAS DOING AND TECHNOLOGY
BUT ONE OF THE KEY THINGS WITH
RECURSION WAS THEY MEET THE
PINNACLE MILESTONE LISTED AS
NASDAQ COMPANY THIS PAST YEAR
WHICH IS A WONDERFUL STORY AND
YES THE GRANT HELPED BUT CHRIS'S
VISION AND WHAT HE'S DONE IS
REALLY PAVED THE WAY TO SUCCESS
OF THE COMPANY.
THEN THE OTHER GRANTEE WE
FOCUSED ON WAS DR. CHANG KIM
BASED IN MILWAUKEE, WISCONSIN.
HE HAD THIS POINT OF CARE
TECHNOLOGY THAT WE WERE FUNDING
FOR DIARRHEAL DISEASE AND WHEN
COVID CAME IN HE SAID I THINK I
CAN PIVOT AND START ADDRESSING
COVID USING THE SAME PLATFORM.
AND THAT IS WHAT HE DID, HE GAVE
A SUPPLEMENT TO DO THAT.
AND CHANG HE IS WORKING
DILIGENTLY TO GET AUTHORIZATION
FOR THE TECHNOLOGY TO BE USED,
HE HAS A PORTAL VAN HE GOES
AROUND MILWAUKEE USING HIS POINT
OF CARE TECHNOLOGY TO GET COVID
TESTS AND HE'S BEEN A GREAT
SUCCESS STORY FOR US AND SHOWING
HOW YOU CAN PIVOT PLATFORM
TECHNOLOGIES AND IN THIS CASE WE
OFFER THE SUPPORT TO GET HIM TO
THAT NEXT CRITICAL STAGE.
GO TO NEXT SLIDE.
FUTURE DIRECTIONS FOR SBER STTR,
FOCUS ON INCREASING
PARTICIPATION OF WOMEN AND
MINORITY OWNED SMALL BUSINESS,
WE WANT TO CONTINUE TO IDENTIFY
SUCCESS STORIES IN WAYS OF
AMPLIFYING OUR COMPANIES
REACHING PIVOTAL MILESTONES THAT
SHOW EFFECTIVENESS OF THE
PROGRAM, PROVIDING EDUCATIONAL
PROGRAMMING TO GRANTEES HOW TO
APPLY TO THE PROGRAM, WHAT WE
ARE LOOKING FOR SPECIFICALLY
HERE AT NCATS AND MORE
IMPORTANTLY, HOW TO LEVERAGE
WHAT WE ARE DOING HERE AT NCATS
IN TERMS OF SCIENTIFIC
PRIORITIES AND SEEING SBIR STTR
AS A WAY OF ALSO GETTING THERE.
SO IN TERMS OF ASPR COUNCIL
MEMBERS I WE ARE ALWAYS LOOKING
FOR OPPORTUNITIES TO DO OUTREACH
FOR THE PROGRAM, IF YOU HAVE
SUGGESTION WE WELCOME THOSE.
AND IDEAS HOW TO LEVERAGE SBIR
WITH OTHER NCATS INITIATIVES AND
PROGRAMS, WE AGAIN HAPPY TO
LISTEN AND I'M GOING TO OPEN IT
UP FOR QUESTIONS.
>> I WAS GOING TO FILE IN
OUTREACH RECOMMENDATIONS --
GOING TO TIME IN OUTREACH
RECOMMENDATIONS.
I DON'T HAVE ANY QUESTIONS.
JUST A COUPLE OF LEADS FOR HER.
>> FANTASTIC.
THANK YOU SO MUCH KELLY,
APPRECIATE THAT.
>> HI, LILY.
ONE OF THE THINGS YOU MIGHT
THINK ABOUT IN EXTENDING THIS IS
REALLY IN THAT ACT AND USING
MENTORS IS TO ALSO PARTNER
TOGETHER WITH THE CTSA PROGRAMS
AND OTHERS WHICH COULD REALLY
USE LOCAL MENTORS SUCCESSFULLY
TRAVERSES BOTH FROM ACADEMIC AND
BUSINESS SIDE THAT COULD WORK
TOGETHER WITH EXTENDING REACH,
ALSO MAKE IT RELEVANT WITHIN THE
OWN COMMERCIALIZATION SYSTEMS
WITHIN OUR ECONOMIC DEVELOPMENT
THINGS WITHIN THE STATES AND
MIGHT HELP THAT SUSTAINABILITY
AS YOU GO PHASE 1 TO PHASE 2 AND
THIS LESSONS LEARNED THAT I
THINK MAKE TEAMS MORE
SUCCESSFUL.
>> THANKS, BECKY.
WE HAVE HAD VERY SUCCESSFUL
ENGAGEMENT WITH VARIOUS CTSAs
THAT HAVE PARTNERED WITH US TO
TALK ABOUT THE PROGRAM.
IN FACT LAST WEEK WE HAD A
WEBINAR WITH A COMPANY THAT WAS
STARTED FROM A K 1 SCHOLAR AT UT
HOUSTON AND HE LICENSED
TECHNOLOGY INTO THE COMPANY.
ALSO PARTNERED WITH AN EXPERT
WITHIN LOCAL CTING ISA.
WE TALK ABOUT HOW TO ENGAGE WITH
THE CTSA PROGRAM IF YOU ARE
SMALL BUSINESS HOW YOU GO ABOUT
DOING THAT.
THE WEBINAR WAS VERY SUCCESSFUL
IN TERMS OF MAKING TALKING THE
STORY HOW TO BRIDGE THE TWO
PROGRAMS, I WOULD LOVE TO SEE
MORE OF THAT OVER TIME AS WELL
TOO.
MAYBE WE CAN TALK SEPARATELY AND
GIVE ME MORE IDEAS ON WHERE TO
GO.
>> LOVE TO, WE CAN TALK OFFLINE.
>> ANY OTHER QUESTIONS FOR LILY?
>> LILY HOW PRESCRIPTIVE CAN YOU
MAKE THE RFAs IF YOU WILL FOR
THESE?
IS IT JUST SORT OF OPEN
EVERYTHING OR CAN YOU DO
TARGETED ANNOUNCEMENTS?
>> ABSOLUTELY WE CAN DO TARGETED
ANNOUNCEMENTS.
YES.
OUR OMNIBUS TOPICS ARE ON THE
BROADSIDE BECAUSE WE WANT TO
CAPTURE AS MUCH AS WE POSSIBLY
CAN WITH THAT.
BUT WE DO TARGET SOLICITATION,
ORDR IS VERY SUCCESSFUL IN
LEVERAGING WHAT THEY -- THEY ARE
DOING USING THE TARGETED
SOLICITATIONS.
THEY HAD A BASKET TRIAL RFA
USING A U MECHANISM ABOUT WE
USED A U 44 MECHANISM ON THE
SBIR AS COMPLIMENTARY WAY OF
GETTING SMALL BUSINESS ENGAGED.
SO IT IS VERY MUCH SOMETHING
THAT WE WANT TO SEE MORE OF
GOING FORWARD AND FIGURING HOW
TO LEVERAGE CURRENT PROGRAMS AND
INITIATIVES USING SBIR STTR.
>> COUPLE OF FOLLOW UPS.
DOES THE END RESULT NEED TO BE A
PRODUCT OR IT -- CAN IT BE A
SUSTAINABLE SET OF SERVICES?
>> TYPICALLY A PRODUCT.
IN TERMS OF SUSTAINABLE SERVICE,
IF THERE IS SOMETHING UNIQUE
ABOUT INNOVATIVE ABOUT THAT
SERVICE, THAT REQUIRES PERHAPS A
PLATFORM OF SOME SORT TO DO, WE
HAVE MANY COMPANIES THAT OFFER
DRUG DEVELOPMENT SUPPORT AND DO
IT AS PART OF SERVICE BUT IT IS
THE TECHNOLOGY THEY USE BEHIND
THE SCENES THAT IS THE
INNOVATION THAT WAS FUNDED BY
SBIR, THAT WOULD BE POSSIBLE.
>> WHERE MY HEAD IS ON YOUR
SECOND QUESTION THINKING BACK TO
JONI'S AUDACIOUS GOALS, LOOKING
AT WHERE THE GAPS ARE THAT MAY
WELL NOT BE EASY TO FILL USING
TRADITIONAL METHODS, SEEMS LIKE
TARGETED ANNOUNCEMENTS MIGHT
OFFER A GOOD OPPORTUNITY.
>> WE ARE UP TO EXPLORING HOW TO
MAKE THAT HAPPEN.
IN SOME INSTANCES HAVING THE
TARGETED ANNOUNCEMENTS IS GOING
TO GET YOU MORE WHERE YOU
IMMEDIATE TO GO, MAYBE THAT IS
EXACTLY IN TERMS OF BRIDGING
THOSE WITH JONI'S GOALS SHE
MENTIONED, SPECIFICITY AROUND
TARGETED SOLICITATION IS REALLY
THE WAY TO GO OPPOSED TO BEING
BROAD.
>> LOVE THAT IDEA.
ANY OTHER QUESTIONS FOR LILY?
OKAY.
HEARING NONE WE HAVE MADE UP
SOME TIME.
ANNA, QUICK PROCESS QUESTION
HERE, SHOULD WE TAKE A BREAK?
OR PROCEED WITH THE LAST BIT OF
THE COUNCIL CONCEPT CLEARANCES?
>> JONI, I THINK FOLKS WOULD
APPRECIATE A SHORT BREAK.
WE DO HAVE TIME, WE ARE 20
MINUTES AHEAD OF SCHEDULE.
SO TEN MINUTE BREAK WOULDN'T BE
PROBLEMATIC AT ALL.
>> SOUNDS GREAT.
EVERYBODY IN TEN MINUTE BREAK.
ALL RIGHT.
LET'S DO IT.
SEE YOU BACK IN ABOUT TEN A
LITTLE AFTER TEN OF 4.
SEE YOU SOON.
SO NEXT UP IS THE CLEARANCE OF
CONCEPTS AND WE HAVE A COUPLE
FOR TODAY AND WE HAVE A COUPLE
FOR TOMORROW.
THE FIRST ONE WITHINOR WILL BE
WITH OFFICE OF POLICY
COMMUNICATIONS AN EDUCATION
CONCEPT AND PENNY BURGOON WILL
KICK OFF AND THEN WE WILL HEAR
FROM JESSICA TAGLE BADGER.
>> THANK YOU, GOOD AFTERNOON,
EVERYBODY.
HERE TO OVERVIEW OFFICE OF
POLICY COMMUNICATIONS AND
EDUCATION.
I WILL ALSO WEAVE INTO A BRIEF
INTRODUCTION ABOUT THE CONCEPT
FOR THE EDUCATION BRANCH.
NEXT SLIDE PLEASE.
THE OFFICE OF POLICY
COMMUNICATIONS AND EDUCATION
OTHERWISE KNOWN AS OPIC PROVIDES
GUIDANCE AND SUPPORT FOR NCATS
ON NUMBER OF ACTIVITIES IN THE
PURVIEW OF SCIENCE POLICY.
COMMUNICATIONS AND OUTREACH.
THE NEWEST BRANCH IS OUR
EDUCATION AN TRAINING BRANCH.
THIS IS A GROUP THAT WAS -- WE
HAD THE ORGANIZATIONAL APPROVAL
PROBABLY AROUND 2017 BUT WE WERE
NOT ABLE TO STAFF THE -- THIS
BRANCH UNTIL 2019.
NEXT SLIDE PLEASE.
NOW, THE GOALS OF THE EDUCATION
BRANCH ARE TO DISSEMINATE AND
DEVELOP EVIDENCE BASED TOOLS AND
BEST PRACTICES.
TO IMPROVE THE UNDERSTANDING OF
TRANSLATIONAL SCIENCE.
AND IN THAT GOAL THEY ALSO HOPE
TO EXPAND AND DIVERSIFY
TRANSLATIONAL SCIENCE WORK
FORCE.
THE BRANCH AIMES TO ACHIEVE
THESE GOLDSBY COORDINATING AND
PROVIDING CENTRAL LEADERSHIP TO
NCATS TRANSLATIONAL EDUCATION
ACROSS THE CENTER.
SO THEY WORK WITH ALL THE CENTER
DIVISIONS AND OFFICES, IN
DEVELOPING NEW INITIATIVES LED
BY THIS BRANCH AND LEADING
CENTER WIDE AND ALSO LEAVE A
CENTER WIDE COMMITTEE FOCUSED ON
TRANSLATIONAL SCIENCE EDUCATION.
HERE IS AN EXAMPLE OF THE
ACTIVITIES THEY STOOD UP IN THE
SHORT TIME THEY HAVE BEEN IN
EXISTENCE. THE 501 COURSE IS
ONLINE COURSE INITIATED BY
EDUCATION BRANCH AND THIS WAS TO
BUILD ON EXISTING EFFORTS BY N
CATS COURSES TO TEACH PRINCIPLES
OF TRANSLATIONAL SCIENCE.
THEY ARE WORKING WITH OUR
COMMUNICATION BRANCH TO IMPRINT
SOME OF THESE IMPORTANT
PRINCIPLES IN TERMS OF BADGES TO
HELP EXEMPLIFY SOME OF THE IDEAS
AROUND TRANSLATIONAL SCIENCE
THAT WE THINK NEED TO HAVE A
PARTICULAR EMPHASIS.
HERE ARE THINGS WE ARE WORKING
ON, THIS IS IN DEVELOPMENT, IT
IS NOT OUT THERE YET BUT THIS IS
AN EXAMPLE OF THINGS WE ARE
TALKING ABOUT IN TERMS OF HOW WE
LOOK AT THE SCIENCE OF
TRANSLATION AND WHAT THE WORK
FORCE, WHAT NEED TO BE EXPERT
GAINED EXPERTISE IN.
WITH THAT THIS WILL LEAD TO NEW
CONCEPT CLEARANCE AND I WILL
HAND THIS OVER TO DR. JESSICA
BADGER EDUCATION BRANCH CHIEF.
>> THANK YOU, THIS IS A PROPOSAL
TO DEVELOP A PORTFOLIO OF
EXTRAMURAL RESEARCH EDUCATION
AWARDS THAT RESULT IN A SUITE OF
ONLINE EDUCATION ACTIVITIES THAT
CENTER TEACHING EFFECTIVE
PRACTICES AND CORE PRINCIPLES OF
TRANSLATIONAL SCIENCE.
WITH KNOW THERE IS A NEED FOR
CURRICULAR ACTIVITIES THAT
DISSEMINATE EFFECTIVE PRACTICES
AND TRANSLATIONAL SCIENCE BASED
ON OUR ACTIVITIES INCLUDING
EXTENSIVE LIT REVIEW AND
EXPERIENCE WITH OUR INTERNAL
TRAINING PROGRAM AND MOST
IMPORTANTLY WE RECEIVED THIS
FEEDBACK FROM THE COMMUNITY.
AS YOU HEARD FROM DR. BERRIGAN
THE EDUCATION BRANCH -- BURGOON
THROUGH DEVELOPMENT OF TWO ON
LION COURSES THAT USE EXAMPLES
OF SUCCESSFUL NCATS LED EFFORTS
TO DEMONSTRATE HOW SPECIFIC
RESEARCH ADVANCES CAN BE
EXAMINED TO OFFER INSIGHT TO
TRANSLATIONAL PROCESS IDENTIFY
COMMON CHALLENGES THAT STYMIE
TRANSLATIONAL PROJECTS AND
SOLUTIONS THAT ARE APPLICABLE
NOT ONLY TO THIS PARTICULAR CASE
BUT ALSO MORE BROADLY TO
RESEARCH INITIATIVES ACROSS THE
TRANSLATIONAL SPECTRUM.
THROUGH EXAMINING CASES N CATS
STAFF ALSO IDENTIFIED THE SEVEN
INITIAL PRINCIPLES FOR EFFECTIVE
TRANSLATIONAL SCIENCE SHARED ON
THE PRIOR SLIDE AND HAVE BEEN
INCORPORATED INTO THESE COURSES.
WE NOW WANT TO EXPAND UPON AND
EXTEND THIS APPROACH THROUGH
STIMULATING ADDITIONAL
TRANSLATIONAL SCIENCE EDUCATION
ACTIVITIES, OR WE CAN CALL THESE
EDUCATION EXPERIMENTS, THAT
FURTHER DEVELOP AND FORMALIZE
TRANSLATIONAL SCIENCE FOR
CONCEPTS APPROACHES AND
STRATEGIES DERIVED FROM THE
EXPERIENTIAL KNOWLEDGE IN THE
FIELD.
WE ALSO WANT TO DO THIS WITH AN
EYE TOWARDS EXPANDING
TRANSLATIONAL SCIENCE WORK FORCE
ENHANCING WORK FORCE DIVERSITY
ENSURING BROAD ACCESS TO
ACTIVITIES AS WELL AS
ENCOURAGING INNOVATION AND
ONLINE TRANSLATIONAL SCIENCE
EDUCATION.
THESE AWARDS FUND FULL CYCLE
FROM DEVELOPMENT TO
IMPLEMENTATION TO EVALUATION OF
THESE EDUCATIONAL ACTIVITIES.
THIS PROPOSAL ALIGNS WITH TWO
NCATS STRATEGIC GOALS, THAT IS
ADVANCING TRANSLATIONAL SCIENCE
BY UNCOVERING TRANSLATIONAL
SCIENCE PRINCIPLES TO CATALYZE
ADVANCES IN HEALTH INTERVENTIONS
SO UNCOVERING PRINCIPLES AND
TEACHING THOSE TO CURRENT FUTURE
MEMBERS OF THE TRANSLATIONAL
SCIENCE WORK FORCE.
AND TO DO THAT, MEET STREAM GOAL
THREE DEVELOPING FOSTERING
INNOVATIVE TRANSLATIONAL SCIENCE
TRAINING AND HIGHLY SKILLED
CREATIVE AND DIVERSE
TRANSLATIONAL SCIENCE WORK
FORCE.
WE REALLY SEE THIS AUGMENTING
CURRENT EDUCATION TRAINING
PROGRAMS.
WE KNOW SOME CURRENT ACTIVITIES
FOCUS ON THE CHARACTERISTICS OF
THE TRANSLATIONAL SCIENCE BEING
SKILLED COMMUNICATOR, DISTINCT
PHASES OF TRANSLATION OR CONTENT
AREAS RELATED TO TRANSLATION.
BUT NOW WE WANT TO ADD TO THIS
HOW DO WE EXPLORE TRANSLATIONAL
SCIENCE BY LOOKING AT THE WHOLE
SYSTEM.
HOW DO WE IDENTIFY THESE
EFFECTIVE PRACTICES AND TEACH
THEM TO OTHERS, AND ARTICULATE
THE BENEFITS OF A TRANSLATIONAL
SCIENCE APPROACH.
AND USING THESE PRINCIPLES COULD
INVESTIGATE A SCIENCE MIND SET.
THIS IS ESTABLISHING
TRANSLATIONAL SCIENCE AS
DISTINCT AREA OF STUDY.
SO I MENTION WE HAVE DONE LIT
REVIEW, I WILL SHARE ABOUT THE
DATA HERE AS IT IS INFORMED OUR
THINKING ON THIS.
BUT WE WERE SPECIFICALLY LOOKING
AT TRANSLATIONAL SCIENCE
EDUCATION WHERE THERE WAS FORMAL
CURRICULUM COMPONENT, EDUCATION
OR COURSE, A WORKSHOP, SEMINAR
SERIES AND THEN WHAT WAS TAUGHT
OR CONVEYED WITHIN THOSE COURSES
WORKSHOPS, ET CETERA.
THIS IS SHOWING YOU CAN IMAGINE
LIT REVIEW FOR TRANSLATIONAL
SCIENCE EDUCATION YOU GET
ARTICLES WHEN YOU NARROW DOWN
SPECIFIC CURRICULUM COMPONENTS
IT BECOMES FEW ARTICLES IN THE
LITERATURE OVER THE SPAN WE LOOK
AT FROM ABOUT 2005 THROUGH 2021.
BASICALLY THE TAKE HOME MESSAGE
IN THESE SLIDES THERE ARE
CERTAIN SEGMENTS OF
TRANSLATIONAL SCIENCE WORK FORCE
WE ARE MISSING OR PEOPLE WHO WE
WANTS BRING INTO THE WORK FORCE.
AGAIN THIS WAS PRE-COVID WE KNOW
A LOT HAPPENED WITH ONLINE
EDUCATION IN THIS TIME.
THERE WAS -- THERE WAS FEW
ARTICLES MENTIONED ANYTHING
ABOUT ONLINE COMPONENT.
FROM OUR LITERATURE RESEARCH.
WE ALSO ORGANIZED ARTICLES BASED
ON FRAME WORK OF TRANSLATIONAL
PRINCIPLES TO SEE WHERE GAPS OR
ADDITIONAL EDUCATION NEEDS COULD
EXIST AND AGAIN THIS SHOWS YOU
THERE'S SOME THINGS THAT WE ARE
TOUCHING ON FREQUENTLY LIKE TEAM
SCIENCE BUT OTHER THINGS WE CAN
BUILD OUT MORE.
SO GIVE YOU SOME OF THE
HIGHLIGHTS FOR THIS PROPOSAL,
WHAT WE ARE PROPOSING IS FUNDING
OPPORTUNITY FOR THE EXTRAMURAL
COMMUNITY TO DEVELOP NEW
EDUCATION OPPORTUNITIES TEACHING
CORE TRANSLATIONAL SCIENCE
CONCEPT, INCLUDING SCIENTIFIC
OPERATIONAL PRINCIPLES KEY
STRATEGIES AND APPROACHES USED
EFFECTIVELY TO ADVANCE
TRANSLATION LOOKING FOR THIS TO
EXPAND AND DIVERSIFY
TRANSLATIONAL SCIENCE WORK FORCE
TO REACH PEOPLE NOT CURRENTLY
REACHING WITH OUR CURRENT
EDUCATION TRAINING PROGRAMS AND
WE REALLY WANT TO STIMULATE
INNOVATION AND EDUCATION CONTENT
DESIGN.
SO WHILE WE WANT THIS AVAILABLE
IN AN ONLINE FORMAT WE ARE NOT
FOCUSED IN ON THIS HAS TO BE
TYPICAL ON LINE COURSE, THERE'S
WAYS THIS CONTENT COULD BE
DELIVERED.
KEY PART OF THIS WILL BE
EVALUATION.
WE WANT THESE EDUCATION
ACTIVITIES EVALUATED TO USE TO
ESTABLISH EVIDENCE BASED FOR
EFFECTIVE APPROACHES IN TEACHING
TRANSLATIONAL SCIENCE CONCEPTS.
SO AT NCATS WE SAY EVERYTHING IS
AN EXPERIMENT WE ARE NOT AFRAID
FOR THIS TO BE AN EXPERIMENT AND
FOR US TO LEARN WHAT WORKS.
THE NEXT SLIDE SPEAKS MORE TO
SOME OF THE THINGS WE WOULD LIKE
TO SEE COLLECTED AS THE
EVALUATION COMPONENT.
THAT INCLUDES DIVERSITY OF THE
PARTICIPANTS IN THESE EDUCATION
ACTIVITIES AND DIVERSITY IN ALL
AREAS TRAINING AND CAREER STAGE,
EMPLOYMENT SECTOR GENDER RACE
ETHNICITY INSTITUTION TYPE
GEOGRAPHY WE WANT TO KNOW
ADHERENCE OR DEGREE OF
PARTICIPATION IN A COURSE, THAT
WOULD BE IMPORTANT.
FOR THEM TAKING THIS TO THE NEXT
LEVEL AND UNDERSTANDING HOW
PEOPLE ARE UTILIZING THIS
KNOWLEDGE AND HOW DOES IT
INFLUENCE THEIR RESEARCH OR
CAREER GOALS GOING FORWARD.
SO WHAT DO WE EXPECT AN OUTCOME
FROM THIS EDUCATION PROGRAM?
BROADEN EXPAND FOUNDATIONAL
TRANSLATIONAL SCIENCE KNOWLEDGE.
DOING THROUGH VARIETY OF ONLINE
EDUCATION ACTIVITIES TO INCREASE
UNDERSTANDING OF TRANSLATIONAL
SCIENCE PRINCIPLES AND EFFECTIVE
APPROACHES AND TRANSLATIONAL
SCIENCE.
WE KNOW ONLINE LEARNING ONLINE
EDUCATION ACTIVITIES ARE HERE TO
STAY, PEOPLE APPRECIATE THE
FLEXIBILITY OF THIS, THEY
APPRECIATE ACCESSIBILITY, SO WE
WANT TO MAXIMIZE ONLINE LEARNING
AND EVALUATE THIS TO LEAD TO
EVIDENCE INFORMED TEACHING
APPROACHES FOR ONLINE EDUCATION
AND TRANSLATIONAL SCIENCE
REACHING DIVERSE AUDIENCE OF
LEARNERS.
WE WANT TO CATALYZE NEW
TRANSLATIONAL SCIENCE CURRICULA.
SO ULTIMATELY THESE EDUCATION
OPPORTUNITIES OR ACTIVITIES
SHOWN TO BE EFFECTIVE COULD BE
INFORM RATED INTO A WIDE RANGE
OF BIOMEDICAL RESEARCH AND
TRAINING, RESEARCH EDUCATION AND
TRAINING PROGRAMS INCLUDING
THOSE FUNDED BY NCATS BUT MORE
BROADLY, ACROSS NIH.
IN SUMMARY THIS PROPOSAL WILL
ADVANCE TRANSLATIONAL SCIENCE
EDUCATION FOR DEVELOPMENT
IMPLEMENTATION AND EVALUATION OF
ONLINE EDUCATION ACTIVITIES
UNDERSTANDING TRANSLATIONAL
SCIENCE PRINCIPLES AN
APPLICATION TO RESEARCH ACROSS
THE TRANSLATIONAL SCIENCE
SPECTRUM.
NOW IS THE TIME TO AGGREGATE
THIS EXPERIENCIAL KNOWLEDGE WE
AMASS AND WE HEARD ABOUT MANY OF
THESE ADVANCES AT OUR TENTH
ANNIVERSARY AND WE KNOW THERE
ARE MORE THAN THAT TO DRAW ON.
TIME TO EXPANDS TRANSLATIONAL
SCIENCE KNOWLEDGE BASE AND
CONDITION SRI TO BROAD RANGE OF
INDIVIDUALS TO EXPAND AND
DIVERSIFY THE WORK FORCE AND
STIMULATE INNOVATION AND
TRANSLATIONAL SCIENCE EDUCATION
AND HOW WE ARE DEVELOPING THAT
FORCE.
ON THE NEXT SLIDE I WILL
TRANSITION TO TAKING QUESTIONS
AND COMMENTS FROM THE COUNCIL,
COUNCIL MEMBERS WE LOOK FORWARD
TO HEARING FROM YOU WHAT IMPROVE
THE INITIATIVE AND OTHER THINGS
WE SHOULD BE CONSIDERING.
>> JESSICA, BEFORE YOU MOVE ON
WE HAVE COUPLE OF COUNCIL
MEMBERS DISCUSSANTS FOR THIS
CONCEPT.
I WILL LET BEBECKY GO FIRST AND
RAJESH AFTER THAT AND HEAR YOUR
THOUGHTS AND WE WILL OPEN FOR
BROADER DISCUSSION.
BECKY, LEAD US OFF?
>> THANKS, JONI.
I LIKE TO START THIS OFF BY
SAYING THAT I'M INCREDIBLY
ENTHUSIASTIC ABOUT SEEING REALLY
LEVERAGE OF BROADER EXTRAMURAL
COMMUNITY, THAT INCLUDES PUBLIC
PRIVATE PARTNERSHIPS AND OTHERS
THAT CAN PULL TOGETHER BOTH
BASIC AS WELL AS MORE ADVANCE
TRAINING IN THE UNDERLYING
PRINCIPLES OF TRANSLATIONAL
SCIENCE AND REALLY SHARING
WITHIN THAT SOME OF THE TOOLS
THAT WE CAN UTILIZE FOR DOING
THAT.
I DO THINK IT IS IMPORTANT
SHOULD BE EMPHASIZED THAT THIS
SHOULD REALLY SUPPORT THAT FULL
SPECTRUM OF TRANSLATIONAL
SCIENCE REALLY FROM TAKING
DISCOVERY TO THE BEDSIDE ALL THE
WAY INTO DISSEMINATION AND
IMPLEMENTATION SCIENCE BUT THERE
ARE COUPLE OF THINGS THAT I
THINK COULD IMPROVE THIS I HAVE
A COUPLE OF SUGGESTIONS TO
RESPOND TO.
THE FIRST I THINK IS THAT AS THE
FIELD OF TRANSLATIONAL SCIENCE
CONTINUES TO EXPAND AND AS WE
GET MORE CLARITY AROUND SOME OF
THE THINGS THAT WILL ADVANCE
GOALS THAT HAD BEEN TALKED ABOUT
TODAY I THINK THAT IS GOING TO
BE CRITICAL THAT ANY TYPE OF ON
LION OPPORTUNITIES ARE KEPT UP
TO DAY SO HAVING THAT
SUSTAINABILITSUSTAINABILITY PLAN REVIEW AN D
UPDATE OF MATERIALS OR SUN
DOWNING THEM AT SPECIFIC PERIODS
OF TIME ARE CRITICAL IN ORDER
FOR THIS TO ACTUALLY HAVE A
SIGNIFICANT IMPACT.
I THINK THE SECOND THING WHICH
YOU MENTIONED IN YOUR SLIDE,
REALLY TO INNOVATE CURRICULAR
YOU KNOWVATION AND I USE THIS AS
A OPPORTUNITY TO SEE WHICH
APPROACHES ARE MOST EFFECTIVE
FOR ADULT LEARNERS IN TERMS OF
ACTUALLY CHANGING BEHAVIOR AND
CULTURE AND BEING MORE ENGAGED
AND TRANSLATIONAL SCIENCE
APPROACHES.
SO ONE OF THE PROBLEMS AS ALL OF
US KNOW WHO CREATED ONLINE
LEARNING, IS THAT IT DOES NOT
FOLLOW THE FIELD OF DREAMS WHICH
YOU TALKED ABOUT EARLIER SO JUST
BECAUSE YOU BUILD IT DOESN'T
MEAN PEOPLE WILL COME.
IT IS IMPORTANT WE THINK ABOUT
REALLY COMING UP WITH MORE
ENGAGING APPROACHES, AND
INNOVATIVE APPROACHES WHETHER
THIS IMPLICATION CASE STUDIES,
EXTENDED MORE IN DEPTH READINGS
ASSOCIATED WITH THIS OR OTHER
TYPES OF ACTIVITIES THAT COULD
BE PURSUED THAT WILL TAKE THAT
LEVEL FROM BASIC TO NEXT LEVEL
MASTERY ALL THE WAY TO ADVANCE.
ONE OF THE THINGS TO THINK ABOUT
THIS IS YOU ALSO WANT TO SUPPORT
OTHER ASSOCIATED ACTIVITIES.
SO DISCUSSION FORMS PEOPLE WHO
ARE INVOLVED IN THIS, OR WAYS OF
STARTING TO DEVELOP A COMMUNITY
OF PRACTICE OR WAYS TO FURTHER
EXPAND THE FIELD.
I THINK THE OTHER THINGS THEN IS
TO THINK ABOUT HOW DO WE REALLY
DRIVE SUCCESS OF THIS.
I THINK IT WOULD BE WORTHWHILE
IN THIS IS RATHER THAN ASKING OR
HAVING A MEETING JUST BETWEEN
THOSE THAT ARE FUNDED IS TO
REALLY THINK ABOUT WHETHER IT
MIGHT BE MORE NIMBLE COST
EFFECTIVE AND GET YOU REALLY
MORE SIGNIFICANT RESULTS IF YOU
CONSIDER THIS AS MORE FORMALIZED
CONSORTIA WHERE YOU BRING
TOGETHER AND MAKE SOME LEVEL OF
CONSISTENCY OF STRUCTURE.
YOU CAN DO THIS THEN THROUGH
PORTAL, DEVELOPMENT OF
EDUCATIONAL ONTOLOGIES THAT
ALLOW PEOPLE TO FIND BOTH
CONTENT THAT BEST MET THEIR
NEEDS AT THE TIME THEY HAD THEM
AS WELL AS MARKETING AND
COMMUNICATION PLANS THAT DRIVE
TO THE RESOURCE AND CONSISTENT
AND ACTIONABLE EVALUATION PLAN
SO YOU ARE COMPARING ACROSS ALL
THE DIFFERENT PROGRAMS THE SAME
KIND OF EVALUATION IN TERMS OF
LOOKING AT LONG TERM IMPACT.
SO DOES THAT CHANGE LEVEL OF
TRAJECTORY OR ENGAGEMENT OF
RESEARCH TRANSLATION, ARE MOVING
THE FIELD MORE RAPIDLY AND ARE
WE BEING MORE EFFICIENT.
I THINK IF YOU THINK ABOUT
EXPANDING THIS PROGRAM BEYOND
JUST THE SERIES OF ON LINE
COURSES TO REALLY SERIES OF
ONLINE ENGAGING ACTIVITIES, AND
LEARNING BETWEEN DIFFERENT
FUNDED PROGRAMS YOU WILL HAVE
GREATER IMPACT ON THE
TRANSLATIONAL SCIENCE WORK FORCE
AND SIMILARLY CAN REALLY THEN
FOCUS ON APPROACHES THAT ALSO
ENHANCE DIVERSITY AND INCLUSION
MULTIPLE PERSPECTIVES AS WELL AS
INDIVIDUALS.
>> THANK YOU, THOSE ARE ALL
FANTASTIC COMMENTS, THINGS WE
CAN REALLY TAKE TO HEART AS WE
CRAFT MORE SPECIFIC LANGUAGE.
I REALLY APPRECIATE YOUR POINT
TO HAVE OUTCOMES WE NEED PEOPLE
TO BE ENGAGED HOW DO WE ENHANCE
THAT ENGAGEMENT.
>> THANK YOU, RAJESH NEXT
PLEASE.
>> SURE.
JESSICA I'M SORRY WE DIDN'T HAVE
A CHANCE TO MEET EARLIER THIS
WEEK YOU CAN CONNECT WITH ME A
COUPLE OF TIMES.
SO I DESIGN/I TEND TO SPEAK MY
MIND SO I WILL HERE.
I'M NOT ENTHUSIASTIC ABOUT THIS,
TELL YOU WHY.
DOES BECAUSE OF MY PAST
EXPERIENCE HAVING TO DO THIS, I
WAS HEAD OF EDUCATION OF
NOVARTIS FOR SEVEN YEARS
MULTI-NATIONAL COMPANY WE
BASICALLY THE ORIGINAL IDEA WAS
DO IT ALL ONLINE, YOU HAVE SEX
CENTERS PUT IT ONLINE, WILL
LEARN DRUG DISCOVERY AND
EVERYTHING WILL BE HUNKY DORY,
ADULTS DON'T LEARN THAT WAY.
THIS IS -- THIS IS NOT THE MODEL
BY WHICH YOU ARE GOING TO GET
THE BIGGEST BANG FOR THE BUCK IN
MY OPINION.
THAT IS WHY THE COURSE IS SUCH
AS THE ONES YOU DEVELOPED
WHETHER THEY ARE ONLINE IN
CLASSROOM SESSIONS SPEAKING WITH
EXPERTS AND WHAT HAVE YOU REALLY
CASE STUDY METHODOLOGY AND OTHER
THINGS THAT MAKE A DIFFERENCE IS
PREFERRED TO JUST PUTTING THAT
OUT THERE BECAUSE IT IS TOO
PASSIVE.
THE OTHER CONCERN IS ONLINE
COURSE IF YOU PUT THIS RFA OUT
THERE OR WHATEVER APPROACH YOU
TAKE CALL FOR APPLICATION MY
CONCERN IS THE COMMUNITY WILL
JUST DEVELOP A WHOLE BUNCH OF
LECTURES, THEY WILL DO A SERIES,
FIND A BUNCH OF PEOPLE WHO GIVE
SERIES OF LECTURES ONE AFTER
ANOTHER, PART ONCOLOGIST
CLINICIAN TALK ABOUT SOMETHING
AND THEN THAT WILL GET POSTED AS
ONE AND THAT WILL GET POSTED AS
OTHER.
QUALITY CONTROL WILL VARY, THERE
WON'T BE ANY STRUCTURE
NORMALIZATION OF PRINCIPLES THAT
WILL BE TAUGHT IN THERE.
IT WILL BE A BOON TOGGLE, I'M
JUST REALLY PRESENTING YOU THE
OTHER CONCERN THAT I WOULD HAVE
IN PUTTING THIS OUT THERE.
YOU SHOULD THINK CAREFULLY ABOUT
THIS IS WHERE YOU WANT TO INVEST
YOUR RESOURCES IN THE
EDUCATIONAL REALM,S WITH THE
ONLINE ARENA.
I I'M NOT -- I WILL THROW
POSITIVES OUT THERE NOT JUST TO
BE NEGATIVE.
ONE THING THAT ACTUALLY WORK
WHICH TOOK A LOT TO DEVELOP, OUT
THERE AS FREE WARE, I KNOW A
BRAZILIAN GROUP, THEY CALL THEIR
GAME SCREENER, WE DEVELOP A GAME
IN PHARMA, IN THE NOVARTIS
CALLED PHARMA GAME OR SOMETHING
LIKE THAT.
FORGET HOW THEY BRANDED IT BUT
YOU CAN DEVELOP A GAME WHICH IS
WHETHER OUR TEENAGERS DO IS PLAY
A GAME ALL THE TIME, PLAY A GAME
AND LEARN DRUG DISCOVERY THAT
MIGHT HAVE MORE IMPACT AND MIGHT
BE A COOL WAY TO MEET APPROACH
OF CURRENT GENERATION DRUG
DISCOVERY.
IF YOU CAN INCLUDE IT MIGHT BE
INTERESTING TO SEE IF
PROGRAMMERS OUT THERE CAN THINK
ABOUT THE COMPLEXITY OF THE
WIRING DIAGRAM IN LAST SLIDE
MEANT TO HAPPEN FASTER AND TAKE
THAT WIRING DIAGRAM AND FIGURE A
WAY TO DO INPUT OUTPUT CIRCUIT
GAIN YOU CAN THEN AS GROUP
DECISION MAKING MAY TRICK.
SO -- MATRIX.
SO PK IS NOT GOOD.
WHAT THE HELL DO YOU DO NEXT?
THAT'S WHAT IT COSTS ANOTHER
MILLION DOLLARS.
DO YOU HAVE THE MONEY, YOU DON'T
HAVE THE MONEY SO HOW DO YOU
KNOW HUH WHOM TO EMPERIMENT.
YOU COLLECT -- YOU DO A
DIFFERENT EXPERIMENT TAKE HELO
CELLS AND THAT DOESN'T WORK.
WHAT IS NEXT EXPERIMENT?
THAT MIGHT TEACH THEM MORE THAN
A BUNCH OF DRY LECTURES THAT
ESSENTIAL WILL I PUTTING TO ON A
WEBSITE.
SO THAT WOULD BE I WILL STOP
WILL.
TRILLION READING WHAT YOU SAY,
I'M NOT SURE WHETHER THE
COMMUNITY THAT IS GOING TO
RESPOND TO THIS I HATE TO SAY
WILL BE OUR GENERATION.
OURS IS GOING TO (INAUDIBLE)
SOMETHING NOT GOING TO WORK FOR
THE NEXT GENERATION IN ONLINE
SPACE.
>> THANK YOU.
ANYTHING TO RESPOND?
WE WILL OPEN IT UP.
>> I APPRECIATE THE COMMENTS AND
I THINK THERE ARE MANY CAVEATS
HERE AND THINGS TO THINK ABOUT.
ONE OF THE THINGS SO HERE ARE
SOME OF THE CHALLENGES THAT I
SEE.
ONE OF THE REASONS WE DID THE --
WE START WITH THE ONLINE COURSE
WHICH AGAIN MAY -- IS VERY
TRADITIONAL, I HOPE WE CAN DO
THIS IN A LOT OF WAYS.
WAS TO REACH MORE PEOPLE MORE
UNDERGRADS AND THINK ABOUT THOSE
SEGMENTS THAT AREN'T HEARING
TRANSLATIONAL SIEBS OR MIGHT NOT
BE AT A HUB IN A FORMAL TRAINING
PROGRAM.
SO REALLY THINKING HOW DO WE
PUSH TRANSLATIONAL SCIENCE
EDUCATION OUT WILL TO MORE
PEOPLE AND EXPAND THE WORK FORCE
AND DI VERY PHI WHO WE RECRUIT
TO THE WORK FORCE.
THAT IS ONE PRINCIPLE UNDERLYING
THIS.
IS GETTING TRANSLATIONAL SCIENCE
EDUCATION TO MORE PEOPLE.
I UNDERSTAND SOME OF THE
CONCERNS YOU MAZE RAISE, WITHIN
NIH WE HAVE DIFFERENT WAYS WE
CAN DI SIGN FUND MECHANISMS
WHERE THERE'S GREATER
INVOLVEMENT FROM M CATS STAFF
AND WE CAN HELP SHAPE THINGS
LITTLE MORE AS DR. JACKSON SAID
THROUGH CONSORTIUM PROCESS AND
SETTING CENTRALIZED EVALUATION
MATRIX -- MEASURES AND BEING
ABLE TO HAVE THAT SORT OF I
DON'T KNOW IF I WANT TO USE
OVERSIGHT BUT MORE CENTRALIZED
FUNCTION ACROSS ALL THESE
ACTIVITIES.
WE COULD HELP SHAPE THAT AS
WELL.
SO I UNDERSTAND WHAT YOU ARE
SAYING.
(OVERLAPPING SPEAKERS)
>> THIS MAY NOT FIT INTO YOUR
SCHEME OF THINGS.
SEND THIS OUT AS THINGS FOR HIGH
SCHOOL STUDENTS AND COLLEGE
STUDENTS TO BUILD RATHER THAN
YOUR PI IN SOME UNIVERSITY AND
THEN GIVE HEM THE EXPERTISE THEY
NEED TO LEARN BUT THEY CAN BUILD
WHAT NEEDS TO BE BUILT TO HELP
PEERS LEARN.
I THINK WHAT WE NEED IN THIS
SITUATION IS SOME THOUGHTFUL
EXPERTISE IN EDUCATIONAL
METHODOLOGY THAT WORKS FOR THE
GENERATION THAT WE ARE TRYING TO
TEACH.
NOT THE SAME YOU AND I LEARNED.
WE MAKE THIS MISTAKE ALL THE
TIME, IF WE TAKE IT THE WAY WE
LEARN IT THEY WILL LEARN IT.
I DON'T THINK THAT WORKS.
>> GOOD POINT.
GO I A HEAD.
>> IT IS CLEARLY A SIGNIFICANT
UNMET NEED THERE IS A FAIR
ENOUGH ACTIVITY IN THE SPACE
WHICH IF YOU CAN REACH INTO
CONCEPT OF ONTOLOGY, FRAMEWORK
WHAT HAS BEEN DEVELOPED
INCLUDING IN OUR FORM OF
TRAINING CERTIFICATE WHERE
TRANSLATIONAL ELEMENTS ARE
TAUGHT, THEIR DISEASE SPACE BUT
MIGHT BE GENERALLY APPLICABLE IN
SEVERAL CTSA SITES, TRANSLATION
TRAINING PROGRAMS ONLINE SO THEN
I THINK WOULD BE DELIVERABLE
WHICH WOULD HAVE IMMEDIATE
IMPACT EXTENDING ON RAJESH
CONCEPT, WE ARE DEVELOPING
COMPETITION FOR SPECIFIC NEEDS
TO SOFTWARE DEVELOPMENT FOR
MOLECULAR BIOLOGY DATA MINING
TOOLS.
THIS COULD BE ADOPTED AS WELL
DEFINE EDUCATIONAL PROBLEM AND
YOU LET COMPETITION COMPETE
LARGE COMMUNITY OF DIVERSE
STAKEHOLDERS AND THEN YOU HAVE A
BENCHMARKING PROCESS AND THAT IS
A KEY PIECE TO DEVELOP
INNOVATIVE CREATIVE EFFECTIVE
BENCHMARK,OBVIOUSLY THE COUNTER
COMPETITIONS BUT MIGHT BE A WAY
TO PULL IN UNEXPECTED IN A WAY
THAT YOU ARE NOT CONSTRAINING TO
ONLY ONE OR TWO BUT YOU GET WIDE
SPECTRUM OF SOLUTIONS, YOU CAN
SET UP COMPETITION THAT YOU HAVE
STAGGERED RESOURCE DISTRIBUTION
TO MAIN PERFORMANCE.
WHICH IS OUT AND COULD BE
ENGAGED.
VERY YOUNG COMPETITORS ENTERING
THESE SPACE AND SOMETIMES THEY
ARE NOT TRAINING LEADING EDGE.
>> YOU MADE COMMENTS IN CHAT.
SAY ANYTHING MOTHER?
>> I THINK WHAT YOU DESCRIBE THE
GOAL OF PROFESSIONALIZING
TRANSLATIONAL SCIENCE AS A
DOMAIN OF EXPERTISE IS
NECESSARY.
IF IF GOAL IS TO REACH YOUNGER
GENERATION I AGREE WITH RAGESH
DO AN EXPERIMENT ON THE SIDE AND
GUILTY IT BUT IF YOU ARE GOING
TO GAMEFY IT DON'T DO IT IN A
WEB 2.0, GO ALL THE WAY.
GO ALL IN AND DO IT IN A WEB 3.0
ENVIRONMENT THEN REACH THE
WORLD.
>> MAKE IT VR.
>> AGAIN, I DON'T WANT TO TAKE
UP ALL THE REST OF THE TIME, IT
IS LIKE I WAS JUST TEXTING
BECAUSE I WAS GETTING REALLY
INTO IT BECAUSE I'M INTO THIS
SORT OF THING AND WE BUILD THIS
STUFF.
SO IT JUST HIT A NERVE IF THE
GOAL IS THE YOUNGER GENERATION,
I THINK IT IS WORTH THINKING
CONTENT DELIVERY IN NEW WAY BUT
YOU HAVE BEEN SO THOUGHTFUL WHAT
THAT CONTENT IS.
IT WAS JUST WRAPPING.
>> I WOULD SECOND KELLY, I THINK
THE CONTENT IS NOT THE ISSUE.
IT WAS REALLY ABOUT HOW YOU WANT
TO GET THIS ACROSS.
WHO YOUR AUDIENCE IS.
IF YOUR AUDIENCE IS 30
SOMETHINGs AND BELOW WHO YOU
ARE WANTING TO BE THE NEXT
GENERATION HAVE GROWN UP
DIFFERENTLY THAN WE HAVE.
>> RIGHT.
YOU DID COME ACROSS AS SOUNDING
LIKE A LITTLE BIT LIKE YOU WERE
CRITICIZING THE CONTEMPT.
JUST MY OPINION.
IT IS GOOD YOU CLARIFIED THAT
YOU DIDN'T MEAN THAT.
>> GOOD.
THANK YOU.
KEITH I SEE YOUR HAND THEN WE
WILL LET BECK KY HAVE THE LAST
WORD HERE.
KEITH GO AHEAD.
>> I THINK YOU ARE MUTED.
>> YOU ARE ON MUTE STILL.
>> THANK YOU.
I'M INTRIGUED BY THE VALUATION
COMPONENT AND ESPECIALLY
INTERESTED IN BEING SURE WE WILL
LEARN HOW TO REACH DIFFERENT
AUDIENCES DIFFERENTLY, I DON'T
MEAN THE YOUTH AUDIENT.
THAT IS MADE ALREADY.
I'M THINKING IN TERMS OF
DIVERSITY, IT IS A CHALLENGE FOR
ME AS AN STROBING TORR EVEN IN A
LIVE ENVIRONMENT TO THINK HOW
DID I SAY THAT AND DID I SAY IN
A WAY THAT RESONATES WITH
DIVERSE AUDIENCE BY RACE
ETHNICITY AGE, ET CETERA.
RATHER THAN JUST GETTING BACK
COUNTS OF WHO AND WHAT TYPES OF
PEOPLE WENT THROUGH WHETHER
GETTING BACK GENERAL IMPRESSIONS
I WOULD LOVE TO SEE INNOVATION.
HOW DO WE LEARN HOW TO TAILOR
OUR PEDAGOGUE NO MATTER WHAT IT
IS TOWARD THE DIFFERENT
AUDIENCES.
AND FINALLY VERY USE -- THEIR
USE OF THAT IN THEIR
TRANSLATIONAL WORK AS THEY REACH
OUT, TO A MORE DIVERSE AUDIENCE.
>> WELL TAKEN.
THANK YOU.
BECKY.
>> IT SOUNDS LIKE THERE IS A
CONSENSUS AMONG ALL OF US THAT
TRADITIONAL LECTURE WITH
POWERPOINT SLIDES, PROBABLY AN
INEFFECTIVE STRATEGY FOR
CERTAINLY CAN BE BACKGROUND
MATERIAL BUT IT CLEARLY ISN'T
THE MOST EFFECTIVE WAY I THINK
IF YOU REALLY EXPAND THIS TO
THINK OF HOW CAN WE USE OTHER
ONLINE THAT INCLUDES LOTS OF
DIFFERENT APPROACH AND THINK HOW
THAT ACTUALLY BRINGS ACROSS
PROCESS GUILTYCATION SOMETHING
WE USE A LOT OF.
SOUNDS LIKE YOU DO TOO.
IT IS SIMPLY MORE ENGAGING BUT
BRINGING TOGETHER IN A WAY THAT
YOU HAVE MORE INTEGRATED SYSTEM
AND THEN THE WAY TO BE ABLE TO
SEARCH WHAT YOU NEED AT THE TIME
YOU NEED IT.
WOULD ADVANCE ALONG AS YOU MAKE
IT AN AVAILABLE SOURCE.
>> THANK YOU FOR THAT.
ANY FINAL THOUGHTS HERE TO
RELAY?
>> THIS IS HELPFUL I'M EXCITED
TO HEAR THE THEWS AM AROUND THE
CONTENT PIECE AND SOUNDS LIKE
THE OTHER SUGGESTIONS GO FURTHER
AS FAR AS FOSTERING MORE
CREATIVE APPROACH HERE.
THAT WOULD BE EXCITING FORWARD
THING FOR NCATS TO DO LIKE
ALWAYS.
>> THANK YOU SO MUCH.
WE HAVE A LOT TO THINK ABOUT.
WBEWILL MOVE TO THE NEXT CONCEPT
CLEARANCE, PRESENTATION.
>> WE NEED TO VOTE.
>> NO FURTHER DISCUSSION.
MOTION TO ADVANCE CONCEPT WITH
THEIR COMMENTS UNDER ADVISE.
>> SO MOVED.
>> SECOND?
>> SECOND.
>> ALL IN FAVOR
>> AYE.
AYE.
>> ANY ABSTENTIONS?
NEXT CONCEPT PLEASE.
>> I WANT THE MAKE SURE YOU GOT
THE ONE EXTENSION THERE.
DID YOU GET IT, ANNA?
>> I ABSTAINED BUT I WAS ON
MUTE.
>> GOT IT.
WANT TO MAKE SURE.
>> WE WILL GO AHEAD AND MOVE TO
THE NEXT CONCEPT CLEARANCE.
THIS IS WITH OFFICE OF SPECIAL
INITIATIVES.
I WILL TURN IT OVER TO DAN
TAGLE.
PLEASE TAKE IT AWAY.
>> THANK YOU, GOOD AFTERNOON
EVERYONE, THANK YOU FOR HANGING
IN FOR THE LAST PRESENTATION OF
THE DAY.
I'M DAN TAGLE, DIRECTOR OF
OFFICE OF SPECIAL INITIATIVES
HERE TO GIVE A BACKGROUND ABOUT
THE OFFICE AS WELL AS SOME
BACKGROUND ABOUT THE INITIATIVE.
NEXT SLIDE PLEASE.
BRIEFLY AGAIN, I MENTIONED IN
CLOSE SESSION THE MISSION OF
OFFICE OF SPECIAL INITIATIVES IS
REALLY TO DEVELOP INNOVATIVE
SOLUTIONS THROUGH DISRUPTIVE
TECHNOLOGY AND OTHER
PARTNERSHIPS IN ORDER THE
ADDRESS THE MAJOR TRANSLATIONAL
PROBLEMS THAT THAT PERVADE IN
THE FIELD.
SO THIS IS A LISTING OF PROGRAMS
AND ACTIVITIES WITHIN OFFICE OF
SPECIAL INITIATIVES.
SO THE CONCEPT THAT IS UP FOR
YOUR DISCUSSION AND CONCURRENCE
IS THE -- SO THAT EXTREME STANDS
FOR EXOSOME BASED THERAPIES FOR
REGENERATIVE MEDICINE, THIS WILL
BE A NEW INITIATIVE WITHIN THE
OFFICE BUT I WANT TO MEMBERS OF
THEY JURY IT LEVERAGES PREVIOUS
INVESTMENTS FROM THE NIH COMMON
FUND.
EXTRA CELLULAR RNA COMMUNICATION
INDICATED IN ONE BULLET ABOVE
WHICH LOOKS AT SECRETED RNA
PRIMARILY FOR BIOMARKER THERAPY
DEVELOPMENT.
SO THERE IS FROM COMMON FUND IN
TERMS OF TOOLS AND RESOURCES
DEVELOPED IN ORDER TO ISOLATE
EXOSOMES.
SO NEXT SLIDE.
WHAT ARE EXSOME?
THEY HAVE BEEN RECOGNIZED AS AN
ARTIFACT OR PERHAPS CELL DEBRIS,
IT HAS ONLY BEEN RECOGNIZED
WITHIN THE LAST DECADE OR SO
THAT IT ACTUALLY CARRIES FORTH
AN IMPORTANT FUNCTION WITHIN THE
BODY.
THAT FUNCTION IS NOVEL MESSENGER
FOR INTERCELLULAR COMMUNICATION.
EXOSOMES ARE ESSENTIALLY
DIFFERENT SHADED FROM OTHER
MICROVESICAL MS. THE SENSE
MICROVESICALS FROM PLASMA
MEMBRANE WHEREAS ENDOSOMES ARE
RELEASED TO THE EXTRA CELLULAR
MATRIX WHEN VESICULAR BODIES
FUSE WITH CELL MEMBRANE SO
ENDOSOMAL ORIGIN.
THEY ARE PRETTY SMALL ORGANELLES
THEY ARE ABOUT 40 TO 150
NANOMETERS.
AND CARRY IMPORTANT BIOACTIVE
MOLECULES WHICH ARE IMPORTANT
FOR SIGNALING AS WELL AS FOR
OTHER CELLULAR FUNCTION.
SO THIS BIOACTIVE CARGO INCLUDES
LIPIDS PROTEINS, MESSENGER RNA
AND IMPORTANTLY ALSO REGULATORY
RNA SUCH AS MICRORNA AND LONG
NON-CODING RNAs.
THESE EXSOMES CONTAIN SURFACE
PROTEINS, ALLOWING IT TO TARGET
TO SPECIFIC CELL TYPES TO ALLOW
SPECIFIC COMMUNICATION BETWEEN
DIFFERENT CELLS.
AND DIFFERENT MILIEU.
I THINK WE HEARD FROM TARA
SCHWETZ EARLIER ABOUT ARPA H AND
WANT OF THE INTERESTS IN
MOLECULAR ZIP CODES, WE HAVE
EXOSOMES THAT ARE NOT POPULATED
WITH CELL SPECIFIC OR TISSUE
SPECIFIC MOLECULAR ZIP CODES
THAT ALLOW TO ZOOM IN WITH
SPECIFIC TARGETS.
IT HAS BEEN RECOGNIZED IMPORTANT
AS INTRACELLULAR SIGNALING
MOLECULES EXSOMES CAN BE USED TO
REPAIR TISSUE INHERENTLY IN ITS
OWN OR BECAUSE YOU CAN ACTUALLY
MANIPULATE NOT ONLY SURFACE
MARKERS BUT ALSO MANIPULATE
CARGO, YOU CAN LOAD UP WITH
VARIOUS THERAPEUTIC CARGOES SUCH
AS PROTEINS, GENE THERAPIES AN
VACCINES TO ALLOW SPECIFIC
TARGETING WITH MINIMIZING ANY
UNDUE EFFECTS IN THE TISSUE.
MENTION SLIDE.
NEXT SLIDE.
THIS IS A IN U CONCEPT
CLEARANCE, IT SAYS EXOSOME
THERAPEUTICS FOR REGENERATIVE
MEDICINE, DR. HAPPEL WILL DO THE
PRESENTATION.
SO SHE HAS A Ph.D. IN GENETICS
FROM THEM IT WILL UNIVERSITY
SCHOOL OF MEDICINE, SHE RECEIVED
THE RUTH KIRSTEIN POST-DOCTORAL
FELLOWSHIP AT HOPKINS WORKING ON
MICRORNA REGULATION AS WELL AS
DOING SECOND POST-DOC AT NCI.
SO CHRISTINE WILL BE TALKING A
LITTLE BIT MORE IN DETAIL ABOUT
THIS CONCEPT CLEARANCE.
SO I WILL TURN IT TO YOU,
CHRISTINE.
>> THANK YOU.
THIS IS A NEW CONCEPT THAT WE
ARE PROPOSING FOR NICELY NAMED
EXTREME.
NEXT SLIDE PLEASE.
DESPITE SUCCESS IN REGENERATIVE
MEDICINE MAJOR CHALLENGES ARE
MAIN IN THE FIELD.
STEM CELL TRANSPLANTATION
CURRENTLY MAIN METHOD FOR TISSUE
REGENERATION PROGRESS IS LIMITED
HURDLES INCLUDE RELIABLE CELL
SOURCE TUMOR FORMATION,
INAPPROPRIATE STEM CELL
MIGRATION, IMMUNE REJECTION OF
TRANSPLANTED STEM CELLS,
COMPLICATIONS DURING SURGERY AND
POST-OPERATIVE INFECTION.
WITH THIS CONCEPT NCATS PROPOSES
A NOVEL EXSOME BASED
THERAPEUTICS PROGRAM TO CATALYZE
REGENERATIVE MEDICINE CREATING A
THERAPEUTIC SO AS SEEN ON THE
SCHEMATIC ON THE RIGHT EXOSOMES
ARE SMALL ENDOGENOUS MEMBRANE
ENCLOSED CARRIERS.
ABOUT THE SIZE OF VIRUS
PARTICLE.
AND AS DAN SAID THEY CONTAIN
BIOACTIVE PROTEIN LIPIDS AND
NUCLEIC ACIDS, FOR INTRACELLULAR
COMMUNICATION.
EXOSOME BASED COMMUNICATION
OCCURS IN A BIDIRECTIONAL MANNER
AND TAKE PLACE DURING NORMAL
CELL HOMEOSTASIS AND CONSEQUENCE
OF PATHOLOGICAL DEVELOPMENT.
SO THE SCHEMATIC SHOWN HERE
SHOWS SIGNALING BETWEEN DISEASE
CELL AND STEM CELL WHERE DISEASE
CELL IS INDICATING DISTRESS.
THE STEM CELL THEN RESPONDS BY
RELEASING SPECIFIC REPAIR
EXSOMES.
WHAT MAKES THIS CONCEPT PARADIGM
SHIFT IS THAT UTILIZES
NON-LIVING CELLULAR PRODUCT
RATHER THAN STEM CELLS
THEMSELVES TO PROMOTE TISSUE
REGENERATION.
THERAPIES TO PROMOTE TISSUE
REPAIR A WOUND HEALING
TRANSLATED TO THE CLINIC.
EXTREME KNOWLEDGE RESOURCES
DEVELOPED BY COMMON FUND EXTRA
CELLULAR RNA COMMUNICATION
PROGRAM WHICH LOOK AT EXTRA
CELLULAR RNA CARRIERS SUCH AS
EXSOMES, AS CRITICAL MEDIATORS
OF INTRACELLULAR COMMUNICATION.
ALSO BUILD OFF RECENTLY
CONCLUDED COMMON FUND
REGENERATIVE MEDICINE PROGRAM,
AND LEVERAGE EXPERTISE OF NCATS
STEM CELL TRANSLATION
LABORATORY.
NEXT SLIDE.
SO THE OBJECTIVE OF THIS PROGRAM
IS TRANSFORM REGENERATIVE
MEDICINE THROUGH NOVEL EXOSOME
BASED THERAPEUTIC AND TISSUE
REPAIR WOUND HEALING.
AS SOON IN THE SCHEMATIC ON THE
RIGHT THERE ARE TWO WAYS THIS
CAN BE ACHIEVED.
THE PERSON HAS DIRECT EXOSOME
THERAPY WHICH UTILIZES EXOSOMES
ISOLATED STEM CELLS STORED AS
OFFICE THERAPY TO BE INFUSED IN
PATIENTS.
SO AN EXAMPLE OF DIRECT EXOSOME
THERAPY, WE FOUND RECENT
PUBLICATION WHERE THEY
DEMONSTRATED ENDOTHELIAL CELL
DERIVED EXOSOMES WERE ABLE TO
INDUCE CARDIAC TISSUE REPAIR,
FOLLOWING ISCHEMIC REPROFUSION
INJURY BY SUPPLEMENTING NEARBY
CELLS BY PROTEINS WITH CRITICAL
CELLULAR PROCESSES.
THE SECOND WAY THIS CAN BE
ACHIEVE IS THROUGH DESIGN
EVERYBODY EXOSOMES WHICH IS
PERSONALIZED ACCORDING TO
INDIVIDUAL PATIENT NEEDS.
THIS MEANS STEM CELL CELLS ARE
HARVESTED FROM PATIENT EXPANDED
AND MODIFIED BASED ON NEEDS
FOLLOWED BY GENERATION OF
EXOSOMES THAT ARE INFUSED BACK
INTO THE SAME PATIENT.
MODIFICATIONS INCLUDE GENETIC
MODIFICATION, ADDITION OF
THERAPEUTIC SMALL MOLECULES OR
CELL SURFACE RECEPTORS FOR
SPECIFIC EXOSOME TARGETING.
WE ANTICIPATE EXTREME ENCOMPASS
KEY AREAS OF EXOSOME
THERAPEUTICS.
DIRECT EXOSOME THERAPY, WOULD BE
MORMORE ACHIEVEABLE, DESIGNER
EXOSOMES PUSH ENVELOPE IN WHAT
CAN BE ACHIEVED.
IMPLEMENTATION OF THIS CONCEPT
INCLUDES FOCUS ON IND ENABLING
THERAPEUTICS AND ENTAIL EARLY
ENGAGEMENT WITH THE FDA TOWARD
REGULATORY APPROVAL.
UTILIZE A MILESTONE DRIVEN
APPROACH TOWARD THESE
THERAPEUTICS TRANSLATED TO
CLINIC, A CONSORTIUM OF FUNDED
INVESTIGATORS TO FACILITATE
SHARING RESOURCES AND EXPERTISE.
METRICS INCLUDE DEMONSTRATION OF
ABILITY TO REGENERATE DISEASE
ORGANS, FROM MANY HUMAN
DISORDERS AND THE VALIDATION OF
THESE THERAPEUTICS BENEFITS IN
VIVO OR EXVIVO.
SO THIS WOULD INCLUDE A
KNOWLEDGE OF SPECIFIC STEM CELL
DERIVED EXOSOME TO ENDEUCE
REPAIR AND WOUND HEALING AT
SPECIFIC TARGET ORGANS.
FINALLY WE EXPECT THAT THE
RESEARCH ACTIVITIES AND RESULTS
SPUR NUMBER OF COMMERCIAL
ACTIVITIES BEYOND INITIAL NCATS
INVESTMENTMENINVESTMENT.
NEXT SLIDE.
IN SUMMARY, EXOSOMES REPRESENT
THE THERAPY DUE TO NATURAL
FUNCTION AS MEDIATORS OF TISSUE
REPAIR AND WOUND HAILING.
NCATS PROPOSES THIS CONCEPT TO
TRANSFORM REGENERATIVE MEDICINE
APPROACHES THROUGH THESE NOVEL
EXOSOME BASED THERAPEUTICS,
CREATING A BUT THERAPEUTIC
PARADIGM.
THIS CONCEPT WOULD DEVELOP CELL
DERIVED HE CANSOME BASED
THERAPEUTICS FOR REGULATORY
APPROVAL AND TRANSLATION INTO
THE CLINIC TO THE BENEFIT OF
PATIENTS. WITH THAT WE WILL
BRING UP OUR QUESTIONS FOR
COUNCIL FOR DISCUSSION.
THANK YOU.
T
>> WE HAVE A COUPLE OF
DISCUSSANTS.
ANNIE WOULD YOU LIKE TO GO
FIRST?
>> I DON'T KNOW THAT I CAN
COMMENT ON THE SCIENCE ITSELF
THAT WAS DESCRIBED BUT I THINK
ONE OF THE THINGS AS I WAS
READING THIS PROPOSAL OVER THE
WEEKEND STRUCK BY WAS FIRST I
APPRECIATE THE INNOVATION NATURE
OF PERSONALIZED MEDICINE.
ONE OF THE THINGS THAT WE TALKED
ABOUT HERE ALREADY TODAY WE
TALKED ABOUT PREVIOUSLY, WAS AS
YOU ARTICULATED THE METRICS
DEMONSTRATION ABILITY TO REPAIR
DISEASED ORGANS FROM HUMAN
DISORDERS AND VALIDATION OF
THERAPEUTIC BENEFITS.
AND I JUST WONDERING IF SPECIFIC
TO THIS PROJECT AND PROGRAM IF
WE SHOULDN'T BE THINKING ABOUT
NCATS CHARGE AS BEING BEYOND
JUST REGULATORY APPROVAL AT THE
FDA?
BUT THINKING ABOUT DECISIONS NOW
HAPPENING WITHIN CMS.
AND HOW IF YOU THINK ABOUT
CONSORTIUM YOU BRING TOGETHER
STAKEHOLDERS AND BUILDING ON THE
PARADIGM YOU ARE THINKING ABOUT
WHAT THE IMPLICATIONS FOR THIS
WILL BE WITHIN THE ENVIRONMENT,
HOW -- WHAT OUTCOMES YOU ARE
LOOKING FOR AS YOU STRUCTURE
THIS TO SHOW THAT BENEFITS IN
VIVO EXVIVO ARE BETTER THAN WHAT
IS AVAILABLE NOW REIMBURSED
SUCH.
SO JUST SOME THINGS FOR
CONSIDERATION AS YOU ARE
BUILDING OUT THE SCOPE OF THIS
AND WE ARE THINKING WHICH
PARTNER SO THAT WOULD BE
DEPENDENT WHICH DISEASE
COMMUNITIES WHETHER YOU ARE
THINKING ABOUT MEDICARE
STRUCTURE OR MEDICAID STRUCTURE,
OR MORE COMMERCIAL ENTITIES.
SO JUST SOMETHING TO THINK ABOUT
MORE BROADLY.
AND THEN OBVIOUSLY DATA SHARING
COMPONENTS WHICH WE ALSO TALK
ABOUT WOULD BE INCREDIBLY
IMPORTANT TO CONSIDER.
THOSE WERE MY ONLY COMMENTS ALSO
EXTEND MORE BROADLY TO OTHER
PROGRAMS WE ARE CONSIDERING AS
WELL.
WE ARE THINKING ABOUT ACCESS
NOW.
NEVER TOO EARLY.
WE ARE THINKING ABOUT PROGRAM
AND HA IS A FANTASTIC WAY TO
KEEP OUR EYE ON THAT BALL.
I APPRECIATE THAT COMMENT.
>> VERY INSIGHTFUL QUESTIONS AND
THINGS WE WILL KEEP IN MIND AS
WE MOVE FORWARD.
THEY ARE VERY IMPORTANT BIG
PICTURE SUGGESTIONS, THANK YOU,
THAT'S HELPFUL.
>> KRISTINA, I THINK I CAN
COMPLIMENT THIS I WILL BE A
LITTLE MORE ON THE TAKING A
MOLECULAR BIOLOGY ASPECT.
I THINK VERY HIGHLY, RNA
CONSORTIUM HAS DONE A TREMENDOUS
JOB IN DEFINING ALL THE METHOD
LOGICAL CONSTRAINT IN THAT
SPACE, AND ASSETS LEVERAGE --
OBVIOUSLY WE HAVE SEEN SPECIFIC
SUCCESSES WITH THESE TYPE OF
INTERVENTIONS, FROM BASIC
SCIENCE APPROACH, MY CONCERN
STRATEGIES THAT IT IS BLACK BOX
APPROACH.
YOU TRANSFER BIOLOGICAL ACTIVITY
WITHOUT CHARACTERIZATION OF
BIOLOGICAL ACTIVITY INTO VARIOUS
DISEASE SETTINGS.
AND WHY THIS APPROACH CAN BE
SUCCESSFUL A NEPHROLOGIST WHO
HAS DONEPHORESES TREATMENTS AND
STUDIES, IT REMAINS BLACK BOX.
YOU WILL HAVE RESPONDERS AND
NON-RESPONDERS AND IT WILL BE
DIFFICULT OR IMPOSSIBLE TO
DEFINE WHO IS RESPONDING AND FOR
WHAT REASON.
THE SCIENCE HAS MOVED FORWARD AS
WE DISCUSSED EARLIER WE HAVE NOW
AN OPPORTUNITY TO ACTUALLY KNOW
THE BAR CODES, YOUR VESICLES ARE
HEADING TOWARDS.
YOU CAN SELECT AND DESIGN YOUR
VESICALES, YOUR VESICLE SORT AND
TYPE GETS PREDEFINED AND
CONTENTS CAN BE DEFINED AS WELL.
WHILE THIS MIGHT BE NOT IN SCOPE
OF THE SPECIFIC OPPORTUNITY I AT
LEAST WOULD DESIGN OPPORTUNITY
SUCH THAT THIS MORE TARGETED
APPROACH CAN BE DEVELOPED OUT OF
CLINICAL IMPLEMENTATION STRATEGY
SO WILL IS A AN ARM BUILT IN
WHEN THE VESICALES ARE
TRANSFERRED AND DEFINED
RESPONSES WHICH THE TECHNOLOGIES
I THINK ARE THERE NOW BASED ON O
CONSORTIUM EFFORT YOU CAN
DAPTURE WHO IS RESPONDING AND
WHAT VESICAL -- CAPTURE WHO
RESPONDING AND WHAT VESICLE
RESPONDS TO MENTION STAGE
TOWARDS MOLECULAR DEFINED
INTERVENTIONS.
OBVIOUSLY VESICALLY TARGETING
TRANSCRIPTS WE HAVE SOME OF
THOSE INTO OUR SHOULDERS
RECENTLY.
AND THEY HAVE BEEN MAJOR BREAK
THROUGH AND FULL DISCLOSURE WE
ARE WRITING WITH SUCH ENTITIES
TO TARGET VESICALES TOWARDS
SPECIFIC CELLS AND SPECIFIC
DISEASES.
SO IN A SINGLE MOLECULE DEFINE
APPROACH ALREADY MOVING FORWARD
BUT YOU STRATEGY MIGHT BE
INTERMEDIATE SPACE WHERE YOU CAN
UNDERSTAND WHERE BIOLOGY
LEVERAGING PRINCIPLE TO COMMENT
TOWARDS MOLECULAR CHEMICALLY
DEFINED APPROACH.
SO CERTAINLY MOVE FORWARD.
BUT TRY TO BUILD DISCOVERY
COMPONENT IN TO THE
TRANSLATIONAL ASPECT
INCENTIVIZING.
>> THANK YOU FOR THOSE COMMENTS.
CHRISTINE.
DO YOU WANT TO --
>> YEAH.
THANK YOU FOR THOSE COMMENTS.
I THINK YOU ARE CERTAINLY RIGHT
IN THAT RIGHT NOW THESE --
PEOPLE ARE STARTING TO GET TO
THIS AREA AND RIGHT NOW IT IS A
LITTLE BIT LIKE BLACK BOX BUT
THAT IS WHY I THINK NCATS IS SO
WELL-POSITIONED TO DIVE IN AND
MAKE SIGNIFICANT IMPROVEMENTS.
BECAUSE WE ARE LEVERAGING A LOT
OF VERY NEW TECHNOLOGIES THAT
HAVE BEEN DEVELOPED.
SO THE M CATS STEM CELL
LABORATORY THEY ARE WELL -- VERY
WELL EXPERIENCED IN STEM CELL
ISOLATION.
SO WE CAN UTILIZE THEIR
EXPERTISE TO GENERATE WELL
CHARACTERIZED SOURCES OF THESE
STEM CELL EXOSOMES AND UTILIZE
KNOWLEDGE FROM EXTRA CELLULAR
RNA COMMUNICATION PROGRAM AN
EXOSOME LYSE ICE LAKES METHODS
THEY DEVELOPED, TO REALLY
CONTRIBUTE TO MAKING SOME VERY
WELL CHARACTERIZED EXOSOME
SOURCES BY PIECING ALL THESE
DIFFERENT TECHNOLOGIES THAT HAVE
BEEN DEVELOPED SO UTILIZING THE
PRODUCTION OF WELL CHARACTERIZED
STEM CELLS FROM A STEM CELLS
TRANSLATION LABORATORY UTILIZING
EXOSOME ISOLATION TECHNOLOGY
FROM EXC CELLULAR COMMUNICATION
PROGRAM AND ALSO SOME OF THE
RESOURCES FROM THE REGENERATIVE
MEDICINE SONOR CONSORTIUM SO IT
IS THIS WHEN YOU FIT PIECES
TOGETHER I THINK REALLY
EVERYTHING IS THERE TO HELP A
MAKE LESS A BLACK BOX AND MORE
WELL DEFINED.
EXACTLY WHAT THE MECHANISM
ACTION IS FOR THESE EXOSOMAL
THERAPIES SO WE UNDERSTAND
EXACTLY WHAT IS TAKING PLACE AND
WHAT IS DERIVING THE THERAPEUTIC
EFFECTS.
>> IF YOU MAKE SURE THE ACTIVITY
IS NOT ONLY LOOKING AT CLINICAL
EFFICACY BUT ALSO BUILT IN
SPECIFIC AIMS TOWARD TO BUY
GENERATING EFFICACY SIGNALS ALSO
ALLOWING TO UNDERSTAND WHICH
MOLECULAR COMPONENT EFFICACY IS
TARGETING TO.
THAT IS MY MAIN COMMENT.
>> GREAT COMMENT.
THANK YOU.
SO
>> SO WE WILL OPEN FOR
DISCUSSION.
I SEE MARSHALL AND RAGESH.
>> ONE OF MY COLLEAGUES IS DOING
WORK IN THIS.
WHAT IS IT ACTUALLY DOING?
IT GETS HAND WAVING QUICKLY.
WHAT ARE THE COMPONENTS HAVING
THERAPEUTIC EFFECT?
WE DON'T KNOW.
THERE'S SOMETHING THERE AND
THINGS LIKE THAT ONE THING I
MIGHT SUGGEST ANYWAY, IS THINK
ABOUT PARTNERING WITH ANOTHER
INSTITUTE WHETHER NSF OR
SOMEBODY ELSE, WHO THE DO THE
BASIC SCIENCE ON THIS.
YOU WILL HAVE WELL CHARACTERIZED
EXOSOMES FROM WELL CHARACTERIZED
STEM CELLS, THAT DOESN'T MEAN
YOU KNOW WHAT IS IN THEM.
AND WHICH HAVING BIOACTIVE
PROPERTIES.
SO WHEN WE WERE DOING PEOPLE
TRANSPLANTS, TAKING ONE TO THE
OTHER PATIENTS WERE SEEING
IMPROVEMENT, I DIDN'T DO THOSE
IN GENETICS.
WHEN YOU ASKED WHAT IT WAS
TRANSPLANTED NO ONE KNEW WHAT IT
WAS.
YOU ARE IN A MUCH BETTER
POSITION BUTTONLY RECOMMEND
PARTNERSHIP WITH TO GET A GOOD
ABASIC SCIENCE COMPONENT OF
THIS.
>> SO PEOPLE DON'T THINK I WOKE
UP ON THE WRONG SIDE OF THE BED,
I DO LIKE THIS PROPOSAL AND
WANTED TO SAY SO.
>> THANK YOU, RAJESH.
I CAN JUMP IN, I THINK THAT THE
ISOLATION TOOLS OF EXTRA
CELLULAR RNA COMMUNICATION
PROGRAM HAS DEVELOPED AS WELL AS
THE ANALYTICAL TOOLS.
I THINK ARE REALLY GOING TO BE
ABLE TO ADDRESS A LOT OF THE
BASIC CONCERNS ABOUT MECHANISM
OF ACTION BECAUSE HISTORICALLY
HE IS SMALL VESICALES ARE
DIFFICULT TO PURIFY.
BUT SOME OF THESE TECHNOLOGIES A
LOT ARE MICROFLUIDIC BASED OR --
THERE'S A LOT OF DIFFERENT
TECHNOLOGIES BUILT THROUGH THIS
PROGRAM.
THEN USING THESE TOOLS WILL HAVE
MUCH MORE HOMOGENOUS PRODUCTION
OF THESE EXOSOMES THAT ARE MORE
WELL CHARACTERIZED.
SO I THINK THAT WILL GO A LONG
WAY INTO REALLY ANSWERING A LOT
OF THOSE QUESTIONS MANY TERMS OF
MECHANISM OF ACTION AND WHAT IS
REALLY RESULTING THIS IN
THERAPEUTIC EFFECT.
>> YOU WILL HAVE A PURE SOURCE
DEEP DIVING EVERYTHING -- EVEN
IF SMALL MOLECULES, DOESN'T HAVE
TO BE BIOLOGIC.
GOD KNOWS WHAT IS IN THESE
THINGS.
>> EXACTLY.
ANALYTICAL TOOLS THAT GO WITH
THESE ISOLATION TECHNOLOGIES,
SHOULD REALLY ALLOW US TO
PINPOINT THE EXACT CARGO
RESPONSIBLE FOR THESE
ACTIVITIES.
SO I'M CONFIDENT THAT THE NEW
TECHNOLOGIES WHICH I KNOW ARE
LOT OF THESE TECHNOLOGIES ARE
JUST GETTING TO THE POINT WHERE
BEING PUBLISHED, BUT I THINK
THEY ARE GOING TO BE A VERY
HELPFUL IN TERMS OF MAKING THESE
-- EXOSOMES USEFUL IN TERMS OF
THERAPEUTICS.
>> STEVE WHAT IS CHANGING IS
THAT WE NOW ARE LEARNING ZIP
CODES WHERE THEY ARE HEADED.
AND WHERE WHAT IS DIFFERENT FROM
THREE FOUR YEARS AGO WHERE THE
EXOSOMAL RNA CONSORTIUM HAD TO
BROADEN THE -- CONCERNING THAT,
THAT'S THE OPPORTUNITY YOU
SHOULDN'T MISS.
SO THAT COUPLE THAT IN DIFFERENT
WAYS YOU CAN CAPTURE SIMILAR
COMPONENTS SIMULATING RNAs IF
YOURY ARE ASSOCIATING WITH
INVASIVE -- TO GET THESE IN YOU
CAN CAPTURE THE CELLS IN THE
NEIGHBORHOOD, YOU ARE EXPOSING
TO YOUR EXOSOMES SO CAN
UNDERSTAND DRUG AND OBVIOUSLY
THERE IS A TIME ON CHIP AND N
CATS BEAUTIFUL PLATFORM YOU CAN
HAVE EXVIVO STUDIES TO SEE IF
YOU BUILD THESE PROCESSES UP IN
HUMANS IN VIVO STUDIES YOU CAN
FOLLOW-UP MECHANISTICALLY AS
WELL SO VERY NEATLY CROSS
FERTILIZATION IN MANY ONGOING N
CATS AND COMMON FUND ACTIVITIES.
>> I WANT TO MAKE ONE MORE
SUBSTANTIVE COMMENT BECAUSE OF
SOMETHING SAID SINCE KEN TO
MINE, WHICH IS WE ARE LOOKING
FOR PURIFIED SOURCES TO DO
BIOLOGY, ONE OF THE COOL THINGS
I HEARD MORE RESEASON WILL I IN
THIS ARENA IS THINKING ABOUT
EXOSOMES IN THE CONTEXT OF GUT
BRAIN ACCESS.
SO THE POSSIBILITY THAT THE
EXOSOMES MIGHT BE DERIVED FROM
PARTICULAR BACTERIAL STRAIN IN
YOUR GUT BUT HAS MEANINGFUL
EFFECT IN THE NERVOUS SYSTEM
MIGHT BE OUTLANDISH TO BELIEVE,
ALLOWS YOU A TESTABLE HYPOTHESIS
BECAUSE IT GIVES YOU A SOURCE OF
EXOSOMES THAT YOU CAN ACTUALLY
THEN PERHAPS MAKE IN LARGE
CULTURES PURIFY, IDENTIFY, MIX
AND MATCH, DO THOSE THINGS THAT
MIGHT GIVE YOU MORE TRACTION
INTO WHAT THE UNDERLYING
MECHANISM MIGHT BE FOR
THERAPEUTICS ATTENTION.
>> THE MAIN AREAS FOR
THERAPEUTIC APPLICATIONS RIGHTS
NOW ARE HEART LUNG KIDNEYS
NEURODEGENERATIVE DISORDERS, AND
EVEN SUCH THING WOUND HEALING
AND CANCER SO THOSE ARE THE MAIN
CURRENT APPLICATIONS THE LOW
HANGING FRUIT BUT THERE IS
TREMENDOUS AMOUNT OF
OPPORTUNITIES IN THIS AREA TO
SEE WHAT THIS THERAPEUTICS TO
DO.
AND THE SYNERGISM WITH CHIP,
WITH THAT WE CAN EVALUATE THAT
THERAPEUTIC POTENTIAL AND BREAK
THINGS DOWN A LITTLE BIT MORE.
THERE IS SYNERGY BEAN THOSE TWO
PROGRAMS AS WELL THAT MAKES
EXCITING.
THE TISSUE CHIP PROGRAM DOES
HAVE BLOOD BRAIN BARRIER CHIP SO
THAT WOULD BE A WAY TO LOOK AT
THAT AND ASSESS THAT.
>> THANK YOU SO MUCH CHRISTINE.
I BELIEVE I WILL TURN TO ANNA
NOW AS WE GET CLOSE TIME HERE.
NEED TO TAKE A VOTE.
I REMEMBERED.
THROW IT OVER TO YOU.
>> THANK YOU.
HEARING NO FURTHER DISCUSSION, I
WOULD LICK TO ASK YOUR APPROVAL
FOR THIS CONCEPT, DO I HAVE A
NOMINATION?
>> MOVED.
>> SECOND.
>> SECOND?
>> SECOND.
>> ALL IN FAVOR?
>> AYE.
AYE.
>> ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT THE SECOND CONCEPT OF
THE DAY IS APPROVED.
THANK YOU VERY MUCH.
BACK TO YOU, JONI.
>> THANK YOU SO MUCH ANNA AND
REST OF THE TEAM FOR THE
PRESENTATIONS TODAY.
I THINK THIS IS THE MOMENT WE
JUST WRAP UP OUR FIRST DAY HERE.
AND I WANT TO INVITE YOU TO
PROVIDE ANY COMMENTS OR
QUESTIONS HERE BEFORE WE CLOSE
OUT TODAY.
JUST QUICKLY MAKE SURE I'M NOT
SEEING ANY.
ANY LAST MINUTE QUESTIONS PLEASE
RAISE YOUR HAND.
IN THAT CASE WE WILL RECONVENE
TOMORROW AFTERNOON AT 1 P.M.
EASTERN AND I LOOK FORWARD THE
SEEING YOU BACK THEN.
I DON'T NEED THE GAVEL OUT NOW
BECAUSE WE ARE STILL IN OPEN
SESSION UNTIL TOMORROW AFTER 5.
STAY TUNED WE WILL SEE YOU
TOMORROW, THANK YOU FOR YOUR
ATTENTION AN ENGAGEMENT TODAY.