I'D LIKE TO WELCOME YOU TO THE
PROGRAM FOR TODAY AND BEFORE WE
JUMP IN IT'S ALWAYS IMPORTANT WE
GAVEL INTO THE SESSION AND SO
I'D LIKE TO ASK THAT OUR ACTING
DEPUTY DIRECTOR DR. SCHMITT
GAVEL UP AND OPEN UP THE SESSION
SO CLAIRE OVER TO YOU.
>>HERE WE GO.
>>WELCOME TO OUR 33rd NCATS OUR
FIRST IN-PERSON-HYBRID AFTER
PANDEMIC CONDITIONS.
IT'S GREAT TO HAVE PEOPLE AND
THE VIRTUAL TABLE AS WELL.
THANK YOU ALL FOR JOINING TODAY.
BEFORE WE GET INTO THE AGENDA
AND OTHER ACTIVITIES OF THE
AFTERNOON I WANT TO TURN IT OVER
TO DR. ANNA RAMSEY-EWING TO GO
OVER A FEW OF THE HOUSEKEEPING
ITEMS AS WELL AS LOGISTICS AND
AGENDA BEFORE WE DIVE INTO THE
REPORTS.
ANNA OVER TO YOU.
>>ROLL CALL.
MEMBERS IN ATTENDANCE ARE SURGE
YO -- SERGIO AGUILAR-GAXIOLA.
ROBIN MERMELSTEIN.
AND NOT PRESENT IS MARSHALL
SUMMAR.
THE AGENDA TODAY FIRST OUR
DIRECTOR WITH THE DIRECTOR'S
REPORT.
NEXT WE'LL HAVE A SPECIAL
PRESENTATION OF THE 2024 NCATS
STRATEGIC PLAN AND THEN AFTER
THE BREAK WE'LL OUR PROGRAM
UPDATE FROM THE DIVISION OF
CLINICAL INNOVATION AND FINALLY
WE'LL HAVE A PROGRAM UPDATE FROM
THE DIVISION OF RESEARCH
INNOVATION BY DOCTORS BROOKS AND
TINA URV.
BECAUSE WE'RE USING A HYBRID
MEETING FORMAT THOSE ON SITE
NEED TO USE THE LAPTOP AND VIDEO
AND WITHOUT AUDIO AND THOSE
PARTICIPATING VIRTUALLY ARE FREE
TO SPEAK BUT WE REQUEST YOU USE
THE RAISE HAND OR CHAT FEATURE
AND WAIT TO BE CALLED UPON TO
PROVIDE INPUT.
ALL PARTICIPANTS ALLOW SPEAKERS
TO MOVE THROUGH THEIR
PRESENTATIONS NOTING COMMENTS
AND/OR QUESTIONS AND PRESENT
THEM WHEN CALLED UPON.
MEMBERS MUST ENSURE DURING
HYBRID MEETINGS CONFIDENTIAL
MATTERS ARE NOT INADVERTENTLY
OVERHEARD BY USING THE
VOICE-ENABLED DEVICES.
WE DO NOT ANTICIPATE WE'LL HAVE
ANY SUCH SESSIONS DURING THE
OPEN WE'RE USING THE VIDEOCAST
FEATURE AS AN IT'S OPEN MEETING.
YOU MAY MAKE COMMENTS UNTIL JUNE
THE BY USING THE E-MAIL FUNCTION
ON THE NIH VIDEOCAST WEBSITE
SEND LIVE FEEDBACK BUTTON OR TO
NCATS COUNCIL INPUT AT MAIL AT
NIH.gov.
MAKE SURE WHEN YOU'RE SPEAKING
YOUR CAMERA IS ON SO YOU'RE
SPOTLIGHTED FOR THE REVIEWERS OF
THE VIDEOCAST.
IF YOU'RE NOT ON CAMERA WHAT
HAPPENS IS THE PREVIOUS SPEAKER
REMAINS HIGHLIGHTED.
THE FUTURE MEETINGS ARE AS
FOLLOWS AND THE JANUARY MEETING
IS GOING TO BE A VIRTUAL MEETING
HELD OVER TWO DAYS MORE THAN
LIKELY.
WE'LL HAVE FACE TO FACE MEETINGS
ON MAY 23 AND SEPTEMBER 26 IN
2024.
IN 2025 OUR JANUARY MEETING WILL
BE HELD ON THE 30th AND 31st
ALSO A VIRTUAL MEETING AND MAY
22 AND SEPTEMBER 25, 2025 WE'LL
HAVE FACE TO FACE MEETINGS.
SO WE ARE CONSIDERING THE
MINUTES FROM THE JANUARY 2023
MEETING.
DO I HAVE THE MOTION TO APPROVE
THE MINUTES?
>>MOVE TO APPROVE.
>>SECOND?
>>ALL IN FAVOR?
ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT THE MINUTES ARE
APPROVED.
NOW WE'LL HAVE THE OPPORTUNITY
TO ENJOY THE DIRECTOR'S REPORT
FROM DR. JONI RUTTER.
TAKE IT AWAY.
>>ALL RIGHT.
THANK YOU SO MUCH
DR. RAMSEY-EWING.
WE CAN GO AHEAD AND PROCEED TO
THE NEXT SLIDE AND I'LL GO AHEAD
AND JUMP IN.
AT OUR LAST COUNCIL MEETING I
ENDED MY DIRECTOR'S REPORT WITH
TWO HIGH-LEVEL SUMMARY AND
THOUGHT IT WOULD BE A GOOD WAY
OF CONNECTING THESE REPORTS
TOGETHER.
I'M JUST SHOWING THIS HERE TO
REMIND US WHAT WE TALKED ABOUT
LAST TIME AND I'LL DO THE SAME
AT THE END OF THIS MEETING TO OR
END OF THIS REPORT.
THE LAST TIME IT WAS MY FIRST
MEETING AS THE DIRECTOR OF NCATS
AND WAS DELIGHTED TO PROVIDE
THAT PRESENTATION AT THE TIME SO
I'M GRATEFUL TO BE HERE AGAIN
TODAY TALKING AND WE'LL TALK A
LITTLE BIT ABOUT SOME OF OUR
IMPACTFUL ADVANCES AND I'LL GIVE
SOME EXAMPLES OF THAT AND LAST
TIME WE ALSO PRESENTED THE PLAN
THAT WE WERE UNDERTAKING FOR OUR
STRATEGIC PLANNING SESSIONS.
I WILL BE DELIGHTED TO TALK TO
YOU ABOUT THE ONGOING ACTIVITIES
IN THAT REGARD AND WE'LL HAVE A
SPECIAL SESSION FOR OUR COUNCIL
TO TALK ABOUT THE STRATEGIC
PLANNING WORK WE'RE DOING.
I LOOK FORWARD TO TALKING ABOUT
THOSE AND WE'LL CONTINUE TO MOVE
ONWARD.
WITH THAT I'LL GIVE YOU A SENSE
OF SOME OF THE COMINGS AND
GOINGS AROUND THE HORN AT THE
NIH AS WELL AS NCATS.
IN THE NEXT SLIDE I WANTED TO
GIVE YOU A SENSE OF THE ONGOING
OPENINGS OR SEVERAL LEADERSHIP
POSITIONS WITHIN THE NIH AND
SPECIFICALLY WITHIN THE
INSTITUTES AND CENTERS AS WELL
AS WITHIN THE OFFICE OF THE
DIRECTOR.
AS YOU CAN SEE THERE'S A VARIETY
OF OPENINGS AT OUR CENTRALIZED
I.T. SPACE WITHIN THE NIH.
OUR FOGARTY INTERNATIONAL CENTER
AND THE NIAID THE OPENINGS
WITHIN THE INSTITUTES AND
CENTER.
WITHIN THE OFFICE OF THE
DIRECTOR, WE HAVE OTHER OPENINGS
AS WELL THE DIRECTORS FOR THE
COMMON FUND PROGRAM.
THAT'S SOMETHING WE WORK CLOSELY
WITH SO WE'RE EXCITED TO SEE
THIS SEARCH IS ONGOING AND AS
WELL AS THE OFFICE OF
LEGISLATIVE POLICY AND ANALYSIS.
SO THESE ARE TWO OF THE HIGHER
LEVEL OPENINGS WE HAVE AT THE
NIH.
OF COURSE YOU HAVE LIKELY HEARD
THE NEWS ABOUT THE NIH DIRECTOR
POSITION.
SO GO TO THE NEXT SLIDE PLEASE.
WE'D LIKE TO CONGRATULATE MONICA
BERTOGNOLLI FOR HER NOMINATION
AS THE NEXT NIH DIRECTOR.
SHE CURRENTLY SERVES AS THE
DIRECTOR OF THE NATIONAL CANCER
INSTITUTE.
THERE'S A BIT OF A PROCESS HERE
THAT NEEDS TO BE TAKE PLACE
BEFORE SHE CAN TAKE THE ROLE OF
THE NIH DIRECTOR AND SO THIS IS
LARGELY PENDING THOSE
ACTIVITIES.
HER CONFIRMATION WILL BE BY THE
UNITED STATES SENATE AND SO
UNTIL THOSE PROCEEDINGS CAN
HAPPEN, DR. LARRY TABAK WILL
CONTINUE IN HIS AS SERVING AS
THE NIH DIRECTOR.
THE PROCESS IS FAIRLY LENGTHY IN
WHICH DR. BERTOGNOLI WILL MEET
WITH MEMBERS OF THE DEPARTMENT
AND BARRING OBJECTIONS THERE
WILL BE A HEARING THAT GENERALLY
OCCURS A FEW DAYS AFTER THE
HEARING TAKES PLACE.
THE ONCE CONFIRMED THEN DR. SHE
CAN BEGIN THE ROLE AS THE NIH
DIRECTOR.
THAT'S A WHOLE PROCESS THAT
NEEDS TO TAKE PLACE BEFORE THAT
CAN HAPPEN.
IT'S STILL PENDING AT THIS STAGE
AND UNTIL OF COURSE THAT TAKES
PLACE THE NCI DIRECTOR POSITION
DOESN'T REQUIRE SENATE
CONFIRMATION BUT SOMETHING WE'LL
NEED TO FOLLOW AFTER THE PROCESS
CONCLUDES.
THAT'S GENERALLY THE PROCESS
THAT WILL GO FORWARD.
STAY TUNED FOR ALL THIS.
LET'S TURN TO THE NCATS
ACTIVITIES AND LEADERSHIP ROLES
AND ACTIVE RECRUITMENTS HERE AS
WELL.
SINCE I WAS NOMINATED OVER THE
LAST SIX MONTHS WE HAVE NOW BEEN
ABLE TO MOVE FORWARD SOME OTHER
ACTING POSITIONS THAT HAVE BEEN
OPEN FOR A WHILE OR HAVE BEEN
ENCUMBERED FOR A WHILE BY GROUPS
OR PEOPLE ACTING IN THESE
POSITIONS SHOWN IN RED.
WE HAVE A VARIETY WE'RE NOW
CONTINUING TO MOVE FORWARD IN
ACTIVE SEARCHS AND THERE'S
VARIOUS STAGES OF THIS PROCESS
BUT WE'RE EXCITED TO GET THOSE
MOVING AND TO HAVE A STABLE
CADRE OF PEOPLE WHO ARE IN THESE
LEADERSHIP POSITIONS AS WE MOVE
FORWARD.
SO THIS IS I HOPE A REALLY
INTERIM STATE WE'RE IN NOW AND I
EXPECT WE'LL BE ABLE TO GIVE YOU
MORE INFORMATION AT THE NEXT
COUNCIL MEETING AS TO OUR
PROGRESS IN THIS AREA.
AND WE HAVE A NEW DEPUTY
DIRECTOR IN THE OFFICE OF
SPECIAL INITIATIVES.
GEETHAT COMES TO US FROM THE
NATIONAL INSTITUTE OF HEALTH AND
FROM THE OFFICE OF GENOMICS
COORDINATION AND PROGRAM
DIRECTOR IN THE DIVISION OF
TRANSLATIONAL RESEARCH.
SHE'S ALSO SPENT TIME IN THE
PORTFOLIO ANALYSIS AND POLICY
ANALYST IN THE OFFICE OF
PORTFOLIO ANALYSIS IN THE
DIVISION OF PROGRAM
COORDINATION, PLANNING AND
STRATEGY INITIATIVES.
THAT'S THE DPCPSI PROGRAM I
MENTIONED EARLIER AND PRIOR SHE
WORKED AS A REGULATORY PROJECT
MANAGER IN THE OFFICE OF BIO
STATISTICS AND EPIDEMIOLOGY AND
THE CENTER OF BIOLOGIC RESEARCH.
AND I'VE HAD THE PLEASURE OF
WORKING WITH THE OFFICE OF DATA
SCIENCE AND STRATEGIES DR. SUSAN
GREGURICK AND GEETHA IS THE ONE
WHO HONES THE PROCESS AND IS THE
PROGRAMMATIC LEAD AND WORKS
ACROSS HHS ON THE PROCESS AND
GREAT TO HAVE HER WITHIN NCATS
AND WOULD LIKE TO WELCOME HER TO
THE FOLD.
THE NEXT SLIDE I WANTED TO
HIGHLIGHT A RETIREMENT.
SAM BOZZETTE WAS OUR CHIEF
MEDICAL OFFICER AND HAD
EXPERTISE IN INFECTIOUS
DISEASES.
HE'S HAD A GREAT DEAL THAT
AFFECTED A GREAT DEAL OF HIV
POLICY AND OTHER INFECTIOUS
DISEASES POLICIES AND ECONOMICS
AND HAS DONE WORK IN CLINICAL
TRIALS AND HELPED US STAND UP
OUR OFFICE OF TRANSLATIONAL
MEDICINE AND THIS HAPPENED RIGHT
WHEN THE PANDEMIC HIT BUT AT THE
SAME TIME HIS INFECTIOUS DISEASE
EXPERTISE WAS REALLY INVALUABLE
DURING THAT TIME AND
PARTICULARLY WORKED WITH THE
ACTIVE PROGRAM PRIMARILY ACTIVE
1 ON THE IMMUNOMODULATORS AND
THE NEWER EFFORT CALLED STRIVE
LIKE THE STRATEGIES FOR TREATING
COVID-19 AND ALSO PLAYED A
CRITICAL ROLE IN THE NATIONAL
COVID COHORT COLLABORATIVE DATA
ACCESS PROCESSES AS WELL.
HE RETIRED LAST MONTH AND WE'RE
WORK ON REPLACING HIM IN THE
COMING MONTHS.
AND THEN ON THE NEXT SLIDE ONE
LAST RETIREMENT I WANTED TO
RELAY TO THIS GROUP BECAUSE THIS
IS A SPECIAL ONE.
AS ANTONETTE NEAL HAS BEEN PART
OF OUR COUNCIL AND MANAGEMENT
TEAM AT NCATS WORKING WITH
DR. RAMSEY-EWING IN THE OFFICE
OF THE DIRECTOR OF EXTRAMURAL
ACTIVITIES.
SHE'S BEEN IN THE GOVERNMENT FOR
OVER 30 YEARS AND HAS BEEN WITH
NCATS FROM THE INCEPTION AND HAS
BEEN A KEY MEMBER OF OUR COUNCIL
TEAM AND HAS BEEN BEHIND THE
SCENES AND I JUST WANTED TO
RECOGNIZE HER IN HER RETIREMENT
AND WISH HER WELL.
SHE'S GOING TO BE MISSED.
THANK YOU.
LET'S MOVE ON TO THE NEXT SLIDE.
I HAVE A FEW NEWS AND
ANNOUNCEMENTS AND STARTING WITH
AN OVERVIEW OF THE BUDGET.
TO THE NEXT SLIDE, PLEASE.
THIS IS THE GRAPHICAL
REPRESENTATION OF OUR BUDGET AT
A GLANCE SO TO PEEK.
THE CTSA PROGRAM IS SHOWN MORE
IN THE TEAL COLOR AND THE CTSA
FUNDS THE NATIONWIDE NETWORK OF
RESEARCH INSTITUTIONS WITH
CONSORTIUM RESOURCES AND
COLLABORATIVES AND ALL OTHER
ACTIVITIES SHOWN IN PURPLE.
THIS MAKES UP A THIRD OF WHAT WE
DO AND IT SUPPORTS THE
INTRAMURAL AND EXTRAMURAL
PROGRAMS INCLUDING DRUG
REPURPOSING, TISSUE CHIPS,
DIAGNOSTICS, ETHICS, TRAINING
AND THE SMALL BUSINESS WORK WE
DO AND ALSO HOUSES THE CURES
ACCELERATION NETWORK THAT
STIMULATE THE TRANSFORMATIVE
EFFORTS AND PLATFORM APPROACHES
ESPECIALLY IN THE AREAS OF UNMET
NEEDS AND ENABLING THE PATIENT
CENTRIC INNOVATIONS FOR STUDYING
AND TREATING THE RARE DISEASES
AND UNDIAGNOSED RARE DISEASES.
I WANTED TO GIVE YOU A SENSE OF
WHAT THAT LOOKS LIKE AND OF
COURSE THE BUDGET PROCESS
GENERALLY STARTS IN FEBRUARY
WITH THE RELEASE OF THE
PRESIDENT'S BUDGET AND THAT
HAPPENED EARLIER THIS YEAR.
AND IT STARTS TO GET INTO THE
SWING OF THINGS AROUND THIS TIME
STARTING GENERALLY IN JUNE.
I DON'T HAVE A LOT OF NEWS TO
REPORT TODAY BUT AFTER THE
PRESIDENT'S BUDGET WAS RELEASED
EARLIER THIS YEAR THERE'S A
PROJECTION OF A FLAT BUDGET AND
OF COURSE THAT'S JUST OF THE
BEGINNING OF THE CONVERSATION.
WE'LL WAIT TO SEE HOW THIS GOES
FOR THE REMAINDER OF THE YEAR SO
STAY TUNED AND I HAVE SOME
UPDATES TO GIVE YOU AT OUR NEXT
COUNCIL MEETING.
GOING TO THE NEXT SLIDE.
I WANTED TO GIVE YOU A SENSE OF
WHAT WE'VE BEEN DOING SINCE WE
LAST MET AND THERE'S BEEN A
NUMBER OF ACTIVITIES THAT ALL
RELY DESPITE BEING ON THE HEELS
OF THE PANDEMIC THERE'S NO REST
FOR THE WARY.
WE'RE JUST AS BUSY AS EVER.
ONE OF OUR BIGGER ANNUAL
MEETINGS WE HOST ALONG THE WAY
AS YOU MAY KNOW IS THE RARE
DISEASE DAY AT THE NIH AND DO
THIS WITH OUR PARTNERS AT THE
CLINICAL CENTER AS WELL AND THIS
YEAR WE HELD IT IN PERSON ALONG
WITH A VIRTUAL OPTION.
AND THE VIRTUAL OPTION WAS A
RESOUNDING SUCCESS AND THREE
TIMES MORE PEOPLE WERE ABLE TO
JOIN VIRTUALLY AND HAD A ROBUST
TURNOUT IN PERSON.
WE HAD A FULL HOUSE IN PERSON
TOO.
AND THIS IS REALLY A FANTASTIC
OPPORTUNITY FOR PEOPLE WHO ARE
NOT ABLE TO TRAVEL BUT REALLY
FEEL A PART OF THIS MEETING AND
GIVEN THE SCOPE AND SCALE IT'S
AN INCREDIBLY LARGE UNDERTAKING
FOR US TO DO TO NAVIGATE ALL THE
POSTERS, THE SPEAKING SESSIONS
AND THE PANEL SESSIONS WITH
PATIENTS, WITH ADVOCACY GROUPS,
WITH THE ACADEMIC RESEARCHERS
AND INDUSTRY.
THIS IS REALLY ALL-COMERS TO
TALK ABOUT WHAT WE'RE DOING IN
RARE DISEASE DAY.
THIS IS A HIGHLY COORDINATED
EFFORT WHO THOSE WHO DESERVE A
LOT OF CREDIT FOR THE SUCCESS OF
THIS MEETING.
GOING TO THE NEXT SLIDE AND I'LL
GIVE YOU PICTURES OF SOME OF THE
PANELS THAT WERE TRANSPIRING
DURING THIS DAY.
WE HAD SPECIAL GUESTS BRIAN
WALLACH AND OTHERS WHO JOINED US
AND THE PATIENT ADVOCATES AND
PATIENTS WERE ABLE TO SHARE MUCH
OF THEIR STORY WITH DRUG
DEVELOPMENT FOR ALS AND WERE
ABLE TO HEAR OTHER STORIES FROM
PEOPLE WITH RARE DISEASES AND
TEACHERS TRAINING THE NEXT
GENERATION OF SCIENTISTS AND
PATIENT ADVOCATES AND HEARING
HOW THEY DEVELOPED
COLLABORATIONS WITH SCIENTISTS
AND INDUSTRY AND AT THE END IT
WAS CAPPED OFF WITH INIMPROMPTU
ANNOUNCEMENT OF A DRUG APPROVAL
TO TREAT FREDERICK'S ATAXIA AND
THE DATE FOR THIS PARTICULAR
RARE DISEASE WAS ON RARE DISEASE
DAY AND SURE ENOUGH THEY HEARD
THE NEWS AT THE VERY END OF THE
MEETING AND WERE ABLE TO MAKE AN
ANNOUNCEMENT.
THAT WAS QUITE A STORY BOOK
ENDING FOR A WONDERFUL DAY.
THIS MEETING HAS IT ALL AND I
HOPE YOU CAN HOLD THE END OF
FEBRUARY OF NEXT YEAR FOR RARE
DISEASE DAY AT NIH IN 2024.
LET'S GO TO THE NEXT SLIDE.
WE ALSO HAD THE ASSOCIATION FOR
CLINICAL AND TRANSLATIONAL
SCIENCE MEETING IN PERSON THAT
MET IN D.C. AND THIS ALSO HAD A
ROBUST TURNOUT AND SOME OF THE
HIGHLIGHTS WERE SHOWN HERE ON
THE LOWER LEFT WE HAD A P.I.
MEETING.
YOU CAN'T TELL THERE WERE OVER
60 P.I.s IN THE ROOM BECAUSE
IT'S FOCUSSED ON THESE PEOPLE
BUT HAD A WONDERFUL DISCUSSION
WITH THE P.I.s PART OF THE
DISCUSSION AND HAT A TOWN HALL
FORMAT TO EXCHANGE IDEAS AND
TALK ABOUT CURRENT ISSUES
RELATED TO A VARIETY OF
ACTIVITIES IN CLINICAL TRIALS
AND COLLABORATIONS AND CLINICAL
DATA AND REAL-WORLD DATA AND HOW
IT CAN ENGAGE AND CAPITALIZE ON
THESE SORTS OF OPPORTUNITIES
MOVING FORWARD.
I THINK THIS IS GOING TO BE
SOMETHING THAT WILL STAY AROUND.
BUT IN ADDITION TO THAT THERE
WAS A LOT OF BUZZ AT THE MEETING
AND WAS GLAD TO SEE THE
DIVERSITY AND TRAINEES AND ALL
THE P.I.s COMING TOGETHER TO
TALK ABOUT WHAT WE'RE DOING IN
THE CTSA PROGRAM.
AND WE'VE TALKED QUITE A BIT
ABOUT HOW ARPA-H IS THE NEW KID
ON THE BLOCK SO TO SPEAK.
THEY JOINED THE HHS FOLD
RECENTLY AND WE'VE BEEN ENGAGING
WIN ARPA-H FROM THE BEGINNING.
THEY'RE STILL GROWING BUT WE
JUMPED AT THE CHANCE TO BRING
THE SMALL NEXUS OF THE EARLY
ARPA-H LEADERSHIP TEAM TOGETHER
TO THE NCATS LABS TO DO A TOUR.
IT TURNED OUT ARPA-H IS A GROUP
THAT WILL NOT HAVE AN INTRAMURAL
PROGRAM.
THEY WON'T HAVE LABS ASSOCIATED
WITH THEM.
SO WE WANTED TO GIVE THEM A
SENSE OF OUR CAPABILITIES AND
TRANSLATIONAL SCIENCE WORK WE
DO.
AND I WANTED TO TAKE A MOMENT TO
MENTION DAN TAGLY IN THE ROOM
TODAY IS OUR DIVISION OR OFFICE
OF SPECIAL INITIATIVES DIRECTOR
AND HE'S OUR CENTRAL POINT OF
CONTACT FOR NCATS AND ALL
INTERACTIONS WITH ARPA-H.
WE HAVE THIS DESIGNATED PAINT OF
CONTACT TO ENGAGE IN IDEAS AND
CONVERSATIONS.
I SPECT THERE'LL BE GOOD
OPPORTUNITIES THERE AND WE'LL BE
ABLE TO RELY THOSE TO YOU IN THE
FUTURE AS THEY START TO UNFOLD.
ARPA-H IS STARTING TO FIND THEIR
SEA LEGS AND HAVE SOME
ACTIVITIES THEY'RE ANNOUNCING
GET UNDERWAY.
WE'LL TALK MORE WHAT
COLLABORATIONS AND PARTNERSHIPS
WE CAN CONVENE.
[DISTORTION]
>>THIS WAS THE LAST LAUNCH ME
OF PROGRAM MARCH 14 AND
SEPTEMBER 2 TISSUE CHIPS UP AND
THEY WERE RELATED TO CARDIAC
ORGANOIDS AND THEY'RE ALREADY
BACK ON EARTH AND BEING
EVALUATED AS WE SPEAK.
THIS IS THE QUITE THE MOMENT TO
BE THERE IN PERSON AND TALK WITH
SCIENTISTS AND RESEARCHERS
SENDING THEM UP TO SPACE AND I
LOOK FORWARD TO HEARING MORE AND
TELLING YOU MORE ABOUT PERHAPS
WHAT MIGHT BE NEXT FOR THIS.
BUT MAYBE WE MIGHT HAVE AN
OPPORTUNITY IN SOME WAY TO
PERHAPS CONTINUE THIS WORK AND
IT MAY GO TO THE MOON WHO KNOWS
MAYBE MARS.
I LOOK FORWARD TO RELAYING THE
WORK WHEN WE KNOW MORE.
IN ADDITION TO THAT LAUNCH THERE
WAS ALSO ANOTHER LAUNCH I WANTED
TO MENTION JUST AS EXCITING.
THE NCATS ALLIANCE WAS LAUNCHED
ON MARCH 15 THE DAY AFTER THE
LAUNCH AND THE ALLIANCE IS AN
ORGANIZATION THAT WORKS
INDEPENDENTLY FROM NCATS.
IT'S REALLY THERE TO RAISE
AWARENESS ABOUT OUR EFFORTS THAT
WE'RE DOING, HELP ADVOCATE FOR
SOME OF THE WORK IN
TRANSLATIONAL SCIENCE AND OFFER
WAYS TO IMPROVE HEALTH AND THE
QR CODE IS SHOWN HERE AND
EXCITED TO SEE THE NUMBER OF
GROUPS THAT ARE ENGAGED WITH THE
NCATS ALLIANCE AND THEY'RE ALL
LISTED HERE.
.
RACHEL HEADS THE ALLIANCE AND
HAS EXPERIENCE IN POLICY AND
LEGISLATIVE ACTIVITIES IN BIO
SCIENCES AND IN REGULATORY
PROCESSES ACROSS ALL STAGES AND
THE LIFE CYCLE OF THE PRODUCTS.
WITH THE CO-CHAIR THEY ORGANIZED
THE GROUP TO ADVANCE THE
TRANSLATIONAL SCIENCE ACTIVITIES
THAT WILL HELP BRING TREATMENTS
TO PEOPLE FASTER.
THIS KIND OF AN ORGANIZATION
THAT IS INDEPENDENT OF US YET
CAN WORK WITH US ON KEY MESSAGES
AND GELLING THESE IDEAS ACROSS
LEADERS AND TRANSLATIONAL
SCIENCE WILL BE CRITICAL FOR US
AND OUR CONSENSUS AND I'M
THRILLED TO SEE THE INTEREST AND
ENTHUSIASM FOR THE NCATS
ALLIANCE.
SO IF YOU GO TO THE NEXT SLIDE
WE ALSO INVITED THE NCATS
ALLIANCE TO DO A TOUR OF THE
LABS AS WELL.
THEY CAN SEE FIRST HAND MUCH OF
THE WORK WE DO AT THE NCATS LABS
AND THIS IS ALSO GOING TO BE
CRITICAL IN HELPING RELAY THE
KEY MESSAGES TO THEM AND HELP
THEM DO THE WORK AS WELL.
VERY EXCITED ABOUT THIS.
I ALSO WANTED TO HIGHLIGHT
ANOTHER ACTIVITY WE DO AT ENACTS
AND THIS IS -- AT NCATS AND A
VOLUNTEER ACTIVITY WITH THE
CHILDREN'S INN AND A DINNER IS
ORGANIZED TWICE A YEAR AND THIS
IS JENNY IN THE CENTER THE CEO
AND THEY PLAY SUCH A CRITICAL
ROLE IN BRINGING KIDS AND LIZ TO
THE NIH CLINICAL CENTER FOR
DOING CLINICAL TRIALS AND BEING
INVOLVED IN CLINICAL STUDIES AND
PROVIDE THIS WONDERFUL PLACE
THAT HAS ALL THE COMFORTS THAT
IS A PLACE LIKE HOME.
I CALL THEM ONE OF THE KEY
TRANSLATIONAL SCIENTISTS BECAUSE
OF THIS ACTIVITY THEY DO AND SO
IT'S ALWAYS A PLEASURE TO BE
INVOLVED IN SERVING DINNERS AND
INTERACTING WITH THE CHILDREN SO
I WANTED TO HIGHLIGHT THAT TOO.
ANOTHER KEY ACTIVITY IN TERMS OF
OUR OUTREACH AND ENGAGEMENT IS
WE PUBLISHED A PAPER ON
TRANSLATIONAL SCIENCE PRINCIPLES
THAT INCLUDES THE VISUAL ICON.
WE'RE REVAMPING OUR INTERNAL
NCATS CATEGORIES TO MATCH THESE
KEY CHARACTERISTICS OF
TRANSLATIONAL SCIENCE AND
PRINCIPLES OF TRANSLATIONAL
SCIENCE.
WE HOPE THESE SMALL ITEMS WILL
SERVE TO REMIND PEOPLE OF THE
TRANSLATIONAL SCIENCE APPROACHES
AND KEEP THEM ATOP OF MIND FOR
THE WORK THEY DO.
SO WE EXPECT TO BE ABLE TO HAND
OUT THE MAGNETS TO PEOPLE WHEN
WE GO TO MEETINGS OR SEE
TRAINEES AS WELL.
THIS IS SOMETHING WE LOOK
FORWARD TO AND PART OF OUR
ENGAGEMENT EFFORTS.
I'LL BRIEFLY TOUCH ON OUR
STRATEGIC PLANNING EFFORTS SINCE
THIG WILL BE A DEEPER DIVE IN
OUR NEXT SESSION.
I WANTED TO GIVE A SENSE OF
WHERE WE ARE WITH THE PROCESS
AND OUR STAKEHOLDER ENGAGEMENTS.
WE STARTED THIS IN NOTCH BER AND
WE'RE REALLY MARCHING THROUGH
AND WE'RE DOING AN OVERVIEW OF
THE NCATS ACTIVES AND TALK ABOUT
OUR GOALS AND ASK QUESTIONS WHAT
THEY THINK ABOUT THE GOALS AND
HOW THEY CAN PROVIDE INPUT IN
HOW WE PROCEED IN DEVELOPING OUR
PLAN GOING FORWARD AND WE SEE
THIS AS AN OPPORTUNITY TO GET
THE INPUT FROM STAKEHOLDERS TO
ALL TAKE OWNERSHIP BECAUSE
THAT'S THE WAY WE'LL BE MOST
SUCCESSFUL.
WE'RE STILL AT THE STAGE OF
DOING THESE STAKEHOLDER
ENGAGEMENTS BUT THEN AS WE GO
FORWARD WHAT WE DO IN A VARIETY
OF OTHER ACTIVITIES AND STARTING
TO PULL THIS TOGETHER IN TERMS
OF REQUEST FOR INFORMATION AND
THEN THE STRATEGIC PLAN ITSELF.
SO WE'LL TELL YOU MORE ABOUT
THAT GOING FORWARD.
AND YOU CAN GO TO THE NEXT
SLIDE.
THIS GIVES A SENSE OF ALL THE
ENGAGEMENTS WE'VE DONE TODAY.
I WON'T DWELL ON THIS BUT TO
GIVE A HEADS UP THE NEXT ONE
WILL BE WITH YOU AND WE'LL DO
THAT IN THE NEXT HOUR OR SO.
I LOOK FORWARD TO GETTING MORE
OF YOUR INPUT IN THIS ACTIVITY.
KNOW I WANT TO TRANSITION TO THE
UPDATES IN COVID RESPONSE AND
HAVE A COUPLE STORIES TO REPLAY.
FIRST IS A LOT OF THESE
ACTIVITIES ARE GOING TO CONTINUE
BUT AS YOU KNOW MAY 11 OF 2023
MARKED THE END OF THE COVID-19
EMERGENCY MEDICAL AUTHORIZATION
AND ALL THE KINDS OF COLLECTING
DATA AS EXPIRED BUT FOR NCATS WE
ESTABLISHED OUR OPEN DATA PORTAL
AND YOU CAN GO TO THE PREVIOUS
SLIDE THEY'RE NOT RELIANT ON THE
PUBLIC HEALTH EMERGENCY THE END
OF THE EMERGENCY DOESN'T REALLY
AFFECT THE PROGRAMS.
HOWEVER, FOR THE CLINICAL
TRIALS, THIS MOMENT DOES SORT OF
MARK THE END OF THE CLINICAL
TRIALS.
THE ACTIVE PROGRAM IS ON ITS
TAIL END EXCEPT FOR ONE THING.
THAT'S RELATED TO THE SLIDE
PREVIOUS TO THIS.
FOR THE CLINICAL TRIALS, MOST
THE CLINICAL TRIALS IN THE
ACTIVE PROGRAMS HAVE ENDED WITH
THE EXCEPTION OF ACTIV6 WE'RE
TESTING THE FIFTH OF FIVE DRUGS
AND THE LAST DRUG IS GOING TO BE
ON METFORMIN.
THIS WILL OPEN IN JULY.
AND THIS IS REALLY BASED ON THE
COVID CLINICAL TRIAL RESULTS
THAT SUGGEST ACUTE METFORMIN
TREATMENT PREVENTS LONG COVID
AND WE'LL LOOK AT TIME TO
RECOVERY FROM ACUTE SYMPTOMS AND
WILL ALSO FOLLOW PARTICIPANTS
FOR SIX MONTHS TO SEE IF THEY
HAVE ANYTHING RELATED TO LONG
COVID SYMPTOMS WE CAN EVALUATE
AS WELL.
WE'RE ALSO INCORPORATING A
COMPONENT WITH EVIDENCE TO LOOK
AT LONG COVID RISK ASSOCIATED
WITH METFORMIN USAGE WITHIN THAT
PARTICULAR RESOURCE AS WELL.
WE'LL BE ABLE TO LOOK BOTH AT
THE REAL-WORLD DATA AS WELL AS
THE CLINICAL TRIAL ITSELF AND
IT'S AN EXCITING TRIAL WE'LL END
OUR PROGRAM ON.
THIS IS A SUMMARY OF THE SARS
THERAPEUTICS TESTED AND CURATED
OVER THE COURSE OF THE PANDEMIC
AND HAS THE RELEVANT DATA
ASSOCIATED WITH THAT.
WE'LL CONTINUE TO KEEP THIS
UPDATED AND ADD A VARIETY OF
OTHER FEATURES AS WELL.
I WANTED TO HIGHLIGHT AN
INTERESTING ACTIVITY.
THIS IS A PHONE CALL OR E-MAIL
TO ME EARLIER THIS YEAR AND IT
WAS A DIRECT REQUEST FROM THE
WHITE HOUSE COVID RESPONSE TEAM
AND THEY WERE ASKING A QUESTION
ABOUT PAXLOVID USE AND WANTED TO
KNOW IF USED AT ITS MAXIMUM WHAT
IMPACT WOULD IT HAVE HAD ON
IMPROVING OUTCOMES WITH
COVID-19.
THEY CHARGED US WITH ANSWERING
SOME OF THESE QUESTIONS.
I WANT TO WALK THROUGH WHAT
WE'VE DONE HERE.
OF THE 2.5 MILLION
COVID-POSITIVE ADULTS AFTER THE
DECEMBER 2021 RELEASE OF THE EUA
FOR PAXLOVID.
ABOUT HALF THE FOLKS GOING
THROUGH THE ELIGIBILITY CRITERIA
WERE ELIGIBLE TO RECEIVE IT
BASED ON GUIDELINES FROM NIH AND
THE CDC AND FDA AND THOSE ARE A
PART OF WHAT CONTRIBUTED TO THE
ELIGIBILITY REQUIREMENTS THAT
WENT IN THE BOTTOM NUMBER.
AND THERE WERE APPROXIMATELY 1.3
MILLION ADULTS IN THIS SPACE BUT
OF THOSE A SUBSET OF 1 MILLION
AT THE BOTTOM MET MORE STRICT
CRITERIA FOR TARGET TRIAL
EMULATION IN STUDYING THIS.
THIS IS A WAY TO LOOK AT REAL
WORLD DATA AND THE DESIGN
PRINCIPLES MUCH RANDOMIZED
TRIALS BUT REALLY USING THE
OBSERVATIONAL DATA WE HAVE IN
HAND AND EXPLICITLY TYING THE
OBSERVATIONAL DATA TO SORT OF
WHAT WOULD BE A CLINICAL TRIAL.
IT'S EMULATING THAT IDEA.
THE PURPOSE IS TO IMPROVE THE
QUALITY OF THESE KIND OF TRIALS
THROUGH THE APPLICATION TRIAL
DESIGN PRINCIPLES ESPECIALLY
WHEN THERE'S NOTHING LIKE A
COMPARATOR OUT THERE THAT
EXQUISITES.
-- EXISTS.
WITH THIS WE WERE ABLE TO LOOK
AT THE PAXLOVID USE AND WHAT WE
FOUND IS THERE'S A TEMPORAL
NATURE TO THE ROLLOUT AND THERE
WAS AN INITIAL ROLLOUT OPTION
AND THIS RAPID ADOPTION.
THEN IT STARTED TO STABILIZE.
AT THE MAXIMUM ABOUT 50% OF
PAXLOVID UPTAKE IN CERTAIN
PLACES WAS UP TO 50%.
THAT SEEMED TO BE THE MAXIMAL
CATEGORY.
THERE WERE OTHER HEALTH CARE
RELATED ORGANIZATIONS THAT HAD
VARIABLE LEVELS UTILIZATION OF
PAXLOVID AND SOME WERE QUITE
LOW.
ON AVERAGE IT'S THE RED BAR
HIGHLIGHTED THERE.
ON AVERAGE IT'S BETWEEN 9% TO
15% OF PEOPLE ELIGIBLE GOT
PAXLOVID.
THERE ARE HIGH AND LOW
PERFORMERS IN THE SPACE.
WE WANT TO WALK THROUGH THAT
MORE TO SHOW YOU WHAT WE MIGHT
IDENTIFY.
IF YOU GO TO THE NEXT SLIDE.
THIS FIGURE WAS LOW SCORING OR
NO SCORING AND ON A PARTICULAR
VARIABLE ON THE LEFT-HAND SIDE
AND GREEN MEANS YES OR HIGHER
SCORING.
FOR EXAMPLE, FOR AGE RED IS
YOUNGER AND GREEN IS OLDER AND
FOR RACE FOR WHITE RACE IS NO
AND GREEN IS YES, WHITE.
ZERO IS LESS LIKELY TO GET
PAXLOVID WHEN ELIGIBLE AND RIGHT
IS MORE LIKELY TO GET PAXLOVID.
THIS IS WHETHER A PRESUMED
ELIGIBLE ADULT GOT PAXLOVID OR
NOT AROUND THIS ANALYSIS.
SOME PREDICTABLE VARIABLES LIKE
THE LIKELIHOOD OF GETTING
PAXLOVID INCREASED WITH AGE.
THIS MAKES SENSE.
THEN THERE WERE A FEW COMOR
COMORBIDITIES LIKE BEING
OVERWEIGHT OR OBESE OR HAVING A
SMOKING ILLNESS INCREASED THE
LIKELIHOOD OF PAXLOVID AND THERE
WERE OTHER VARIABLE.
FOR EXAMPLE THE LONGER YOUR OVER
ALL MEDICAL HISTORY INDICATED
YOU WERE MORE LIKELY TO RECEIVE
PAXLOVID RESULT.
THESE WERE SOME OF THE FINDINGS
WE WERE ABLE TO EVALUATE HERE.
AND CONTRIBUTE IT TO
UNDERSTANDING OF THE RESULTS
HASN'T MORE AND THIS SHOWED
PAXLOVID AND THIS IS BASED
AROUND THE 28-DAY MORTALITY
SHOWN ON THE BOTTOM OR REDUCED
HOSPITALIZATION SHOWN ON THE TOP
THERE.
AND SO FOR PAXLOVID AND FOR
PREVENTING HOSPITALIZATIONS AND
DEATH YOU CAN SEE A BIT OF A
REDUCTION IN BOTH
HOSPITALIZATION AND MORTALITY.
THE PANELS ON THE RIGHT-HAND
SIDE SHOW THESE WERE ROBUST
FINDINGS EVEN WITH THE MULTITUDE
OF DIFFERENT SENSITIVITY KINDS
OF ANALYSES AND DIFFERENT
VARIABLES WE LOOKED AT.
THESE WERE QUITE ROBUST IN THIS
SHOWING THE NICE SEPARATION OF
THE EFFECTIVENESS OF PAXLOVID IN
THE REAL WORLD DATA SET.
IF YOU GO TO THE NEXT SLIDE THIS
SHOWS DEATHS PREVENTED AT
INCREASING LEVELS OF PAXLOVID
UPTAKE.
IF WE WERE TO IF WE SENT THESE
OUT WE CAN DO THE PROJECTIONS
BASED ON THE DATA WE HAD.
WE'RE ABLE TO CALCULATE THE
NUMBER OF DEATHS AND
HOSPITALIZATIONS PREVENTED BASED
ON THE NATIONAL DATA FOR COVID
CASES DURING THE FOLLOW UP
PERIOD OF THIS STUDY AND WERE
ABLE TO PROJECT THAT.
WE ESTIMATED A PAXLOVID UPTAKE
AMONG ELIGIBLE PATIENTS
INCREASED FROM 9% AS WAS
OBSERVED IN OUR STUDY TO 50%.
THEN OF THE PEOPLE ELIGIBLE TO
RECEIVE PAXLOVID DURING THE
FOLLOW-UP PERIOD APPROXIMATELY
48,000 AND 135,000
HOSPITALIZATIONS COULD HAVE BEEN
PREVENTED.
THIS IS STRIKING DATA FROM N3C
AND TO PUT IT IN CONTEXT.
I RECEIVED THIS REQUEST FROM THE
WHITE HOUSE ON MARCH 1 AND
DISCUSSED IT WITH MIKE CORILLA
AND ASKED THEM TO PULL THE
DESIGN TOGETHER WITH OUR
INTERNAL TEAM AND PRESENT THE
RESULTS TO THE TEAM IN MID APRIL
AND WE FINALIZED THE SUBMISSION
WHICH SHOULD BE IN META-ARCHIVE
TODAY.
I CAN'T SHOW THE QR CODE BUT
HOPEFULLY IT WILL BE UP SOON.
OUR NCATS TEAM ANSWERED THE CALL
FOR THIS IMPORTANT INSIGHT THAT
WOULD NOT HAVE BEEN POSSIBLE
WITHOUT N3C AND THE REAL WORLD
DATA RESOURCE AND CREDIT TO THE
GROUP ON THE SLIDE.
[LISTING NAMES]
THIS IS A TESTAMENT TOLL CTSA
GROUP AND CTR PROGRAM AND
COLLABORATORS AND THE THOUSANDS
OF PEOPLE WHO MADE IT POSSIBLE.
IT'S A POWERFUL EXAMPLE OF HOW
IMPORTANT THEY CAN BE TO PROVIDE
INSIGHT WE WOULDN'T BE ABLE TO
DO OTHERWISE.
I WANTED TO GIVE YOU A DEEP
SENSE OF THAT PARTICULAR WORK
AND ACTIVITY THAT WE'VE BEEN
DOING.
LET'S GO TO THE NEXT SLIDE.
I ALSO WANTED TO GIVE A SENSE OF
THE OTHER IMPACTFUL STUDIES
BEFORE ENDING THE SESSION.
I WANTED TO HIGHLIGHT THE
TRENDING AND EDUCATION SPACE FOR
YOUNG PEOPLE AND ADVANCING THE
CAREERS OF WOMEN IN BIO
MEDICINE.
WE HAVE A WOMAN SCIENTIST
ADVISERS GROUP AND THEY GIVE OUT
THE NEW INITIATIVE IN 2022 THAT
PROVIDES OUTREACH AND ENGAGEMENT
TO LOCAL COMMUNITIES AND KIDS IN
KIDS GRADE K-12 FOR TALKING
ABOUT SCIENCE AND BRINGING THEM
IN TO LOOK AT LABS AND THIS IS A
WAY TO INCREASE OUR VISIBILITY
WITH THE FUTURE WOMEN
SCIENTISTS.
I WANT TO ALSO NOTE THAT AS MANY
MAY REMEMBER DR. BECKY JACKSON
WAS AN A PART OF OUR NCATS
COUNCIL AND DIED UNFORTUNATELY
LAST YEAR.
THE ACTS GROUP HAS DEDICATED
THEIR TRANSLATIONAL RESEARCH
DISTINGUISHED EDUCATOR AWARD TO
THE MEMORY OF BECKY JACKSON FOR
OUTSTANDING ACHIEVEMENT IN
EDUCATION INNOVATION.
I WANTED TO HIGHLIGHT THE FIRST
PERSON WHO RECEIVE THE AWARD,
CYNTHIA MORE IS RECEIVED THIS
ACHIEVEMENT IN EDUCATION
INNOVATION AND CONGRATULATE HER
FOR THEIR IMPACT IN THE FIELD
WITH THE WORK.
I WANTED TO HIGHLIGHT A FEW
SCHOLARS.
THIS IS CAROLINE HSU LOOKING AT
SARS COV2 IN AND HAS FOUND AMONG
PATIENTS WHO RECEIVED
MAINTENANCE DIALYSIS THERE'S
STILL A HIGH RATE OF COV2
INFECTIONS AND SUPPORTS THE ROLE
FOR MASKING AND PERHAPS OTHER
RECAUTIONS AND CONTINUING HOW TO
CONTINUE THE VACCINES SO THAT IN
CASE THE TIDERS WANE OVERTIME
FOR THOSE VULNERABLE THIS IS
IMPORTANT WORK SHE'S IDENTIFIED.
SHE LOOKED AT THIS POPULATION
AND SAW IT AS A VULNERABILITY
NOR POPULATIONS AND CREATING THE
DISSEMINATION TO PROTECT MOST AT
RISK FROM SARS COV2 INFECTION.
ANOTHER SCOL HOLAR I WANTED TO
HIGHLIGHT IS JILL ROBERTS IS
LOOKING AT MOIA MOIA SYNDROME
WHICH MEANS PUFF OF SMOKE IN
JAPANESE.
THIS IS WHEN BLOOD VESSELS LOOK
LIKE A PUFF OF SMOKE ESSENTIALLY
ON AN ANGIOGRAM.
A FAIRLY RARE CONDITION AND HAS
A MOUSE MODEL WHERE SHE PUTS
COILS AROUND THE BLOOD VESSELS
TO RESTRICT THEM AND CREATE A
MOUSE MODEL.
SHE'S SET TO TEST THIS AND OTHER
TYPES OF COMORBID CONDITIONS
BECAUSE PEOPLE TYPICALLY WITH
THIS DISEASE HAVE AN INCREASED
PREVALENCE OF AUTOIMMUNITY AND
DIABETES AND THERE'S A TEN-FOLD
RATE OF MOYAMOYA DECEASE IN
KENTUCKY AND ABLE TO CREATE THE
MOUSE MODEL AND HOPEFULLY
DEVELOP TREATMENTS FOR THE
DISEASE SHORTLY.
MORE TO COME ON THAT.
THIS IS A STORY OF KIM STINGER
THE ONLY PERSON IN THE WORLD WHO
HAS SOMETHING CALLED COMPLETE
SENSORY NEUROPATHY AND NO SENSE
OR SENSATION AND SOME ARE TRYING
TO HELP KIM UNDERSTAND HER
CONDITION.
THEY'VE CREATED A DOCUMENTARY ON
THE QR CODE AND CBS DID A
PROGRAM AS WELL ON THE LOWER
LEFT CORNER.
IT'S A TWO-MINUTE VIDEO TRAILER.
I ENCOURAGE YOU TO TAKE A LOOK
AT IT AND LEAVE YOU ROOTING FOR
KIM AND PEGGY AND WANTING TO
LEARN MORE ABOUT THE DISEASE AND
IT HAS A BIG IMPACT HOW THE WORK
CAN ALSO INFLUENCE AND SPORT
SCIENCE BECAUSE OF THE HOPE AND
IMPACT IT CAN HAVE ON LIVES FOR
PEOPLE WITH SIMILAR CONDITIONS
AS WELL.
THIS SANE INTERESTING STORY.
ON THE NEXT SLIDE I WANT TO
HIGHLIGHT ANOTHER FOUR-MINUTE
VIDEO.
THE EXECUTIVE DIRECTOR OF THE
HEALTH EDUCATION COUNCIL OF
CALIFORNIA AND MEMBER OF THE
U.C. DAVIS CENTER FOR REDUCING
HEALTH DISPARITIES AND CTSA AND
COMMUNITY ADVISORY BOARD MEMBER
AS WELL.
THIS PROJECT WAS INCLUDED IN THE
NATIONAL ACADEMY PROGRAM ON
ASSESSING MEANINGFUL COMMUNITY
ENGAGEMENT.
THEY RECENTLY RELEASED THESE
ASSESSMENT INSTRUMENTS THAT WILL
BE IMPORTANT FOR THE COMMUNITY
ENGAGEMENT PROGRAMS WE ALL DO.
I WANT TO THANK SPECIFICALLY
SERGIO AGUILAR-GAXIOLA AND THEY
LOOKED AT A FOOD DESERT IN
NORTHERN CALIFORNIA AND FOUND IT
WASN'T THE ISSUE.
IT'S MORE OF A COMMUNITY FEELING
SAFE AND CONNECTED AND THE
ABILITY TO WALK AROUND ON
PAVEMENT THAT IS WELL CARED FOR
TO PUSH STROLLERS AND NAVIGATE
THE COMMUNITY.
THIS IS WHAT IT'S REALLY ABOUT
AND THIS WAS A LOT OF WORK
CONDUCTED THROUGH THIS AND THE
VIDEO'S JUST A COMPELLING STORY
WHY THIS WORK IS SO IMPORTANT.
I WANTED TO GIVE YOU A SENSE OF
THAT.
THE NEXT STOREYY -- STORY IS TH
HEALTH SCIENCES AND INNOVATION
ENTREPRENEUR PROGRAM THROUGH THE
CTSA OUT OF UMAS AND TWO ARE
STUDY A DRUG THAT'S SUPPOSED TO
HELP.
BUPRENORPHINE IS SUPPOSED TO
HELP OPIOID ADDICTION AND
WITHDRAWAL AND WHAT HAPPENS
THOUGH IN PREGNANCY THERE'S AN
ACTIVE METABLITE AND IT'S
INDICATED WHEN TAKEN DURING
PREGNANCY AND REDUCING OR
ELIMINATING THAT BUPRENORPHINE
METABOLITE IS IMPORTANT TO HELP
IMPACT THE NEONATAL OPIOID
WITHDRAWING SIN DOME ISSUE.
WHAT THEY'VE DONE IS ESSENTIALLY
CREATED A FORM OF BUPRENORPHINE
AND THE FORM DOESN'T GET
METABOLIZED SO IT'S A WAY FOR
PREGNANT INDIVIDUALS TO BE ABLE
TO UNDER GO WITHDRAWING MORE
SAFELY AND NOT AFFECT THE AS
MUCH.
THEY'VE DIN THE WORK IN MOUSE
MODELS AND LOOKING TO FOREIT
FORWF --
MORE IT FORWARD.
AS THE EXCITING.
SPEAKING OF COMMERCIALIZATION I
WANT TO MENTION ONE MORE IN THIS
AREA.
THIS IS ONE OF THE SPIR SUCCESS
STORIES I MENTIONED.
A SMALL BUSINESS PROGRAM AND
THIS IS REALLY A GREAT RESOURCE
FOR SMALL BUSINESSES AND THE
STIR PROGRAM.
THIS PARTICULAR GROUP IN 2018
AND 2021 PROVIDED ABOUT $2
MILLION OF SPIR GRANTS TO
DEVELOP A PROTOTYPE DEVICE FOR
MOLECULAR STRUCTURES IN A
STREAMLINED WAY AND AUTOMATED
WAY AND IT TO THE CLIN IKDZ
FASTER.
-- CLINICS FURTHER AND THE
REFINE THE DEVICE AND HOOKED IT
UP WITH MASS SPECTOMETRY AND
THEY WERE BOUGHT AS PART OF A
$40 MILLION DEAL.
AND THEY'RE MOVING FORWARD IN
THE FIELD AND A WANTED TO
HIGHLIGHT THAT AS WELL.
TO ROUND OUT THIS IS THE LAST
GROUP -- THING I WANTED TO TALK
ABOUT BUT THE LAST IS AN
INTERSECTION BETWEEN THE
BIOLODGICS AND RARELIEDS.
PULLING IT TOGETHER AND MOVING
IT IN THE GENE THERAPY SPACE.
I'M GIVING A TEASE ER OF WHAT
YOU'LL HEAR FROM PJ BROOKS.
WE LEAD EFFORTS AND THE MAIN
PROGRAMS ARE SHOWN HERE AND THEY
SPAN THE CLINICAL TRIAL
READINESS AREAS AND RANGE FROM
SORT OF EARLY ON IN DEVELOPMENT
STAGES LIKE THE EDITING PROGRAM
AND SOMEWHERE IN THE MIDDLE THE
BESPOKE GENE THERAPY CONSORTIUM
AND THE PLATFORM VECTOR GENE
THERAPY PROGRAMMING ON TRACK TO
PERFORM A CLINICAL TRIAL NEXT
YEAR AND IN NCATS WE TALK ABOUT
THE SCIENTIFIC AND OPERATIONAL
CHALLENGES WE HAVE.
AND THESE ARE THE KEY THINGS
WE'RE TRYING TO ADDRESS IN TERMS
OF BOTTLE NECKS AND THE
PRINCIPLES WE'RE TRYING TO PUSH
AND THIS IS REALLY ADDRESSING
BOTH THE SCIENTIFIC AND
OPERATIONAL BOTTLE NECKS BUT
I'LL HIGHLIGHT THE OPERATIONAL
PIECE AND THE PIECE I WANT TO
MENTION HERE.
WE INITIATED THE COLLABORATION
WITH NIGRI AND NINDS AND THE
CLINICAL TRIAL CENTER TO START
THE PROGRAM INTENDED TO DEVELOP
GENE THERAPIES FOR FOUR RARE
DISEASES.
AS A UNIQUE PART OF THE PROGRAM
WE SET OUT TO PUBLIC SHARE THE
REGULATORY EXPERIENCE WE GAINED
AT DIFFERENT STAGES ALONG THE
WAY FOR IMPLEMENTING THE PAGT
PLATFORM.
EARLIER THIS YEAR THE TEAM
OBTAINED ORPHAN DRUG DESIGNATION
AND RARE PEDIATRIC DISEASE TO
TREAT PROPIONIC ISCHEMIA AND
WE'RE GIVING AN INCREASE IN THE
ORE FIN DRUG AND RARE PERIOD
TRICK DISEASE DESIGNATIONS FOR
GENE THERAPY ESPECIALLY FROM
SMALL COMPANIES.
WE FEEL COMPELLED TO MAKE THE
TEMPLATES WE'VE DONE IN OUR
CONVERSATIONS WITH THE FDA
AVAILABLE TO THE PUBLIC.
WE'RE PROUD OF THIS WORK AND A
WANT TO THANK THOSE WHO LED THE
EFFORT AND COLLABORATING WITH
OUR NCATS TEAM AND STRATEGIC
ALLIANCES AND OTHERS IN DIDRI.
COULD BE CONGRATULATIONS TO THEM
FOR THE GAME CHANGING KIND OF
WORK.
I'LL STOP THERE AND OPEN IT UP
IN CASE ANYBODY HAS ANY
QUESTIONS.
THIS IS A SUMMARY OF WHAT WE
TALKED ABOUT AND THE NOMINATION
OF THE NIH DIRECTOR AND THE
ROCKET LAUNCHES AND LOTS OF
IMPACTFUL ADVANCES WE'RE VERY
EXCITED ABOUT AND THE STRATEGIC
PLANNING DISCUSSIONS.
UP NEXT YOU'LL HEAR DR. EWY
MATHE.
I'M HAPPY TO QUESTIONS AND NOTE
WE HAVE SPECIAL TIME AT THE END
OF THE DAY GIVEN ALL THE UPDATES
TO CONTINUE THE DISCUSSION THEN
AS WELL SO WE CAN STAY ON TRACK.
>>I SEE YOU.
>>THAT'S A GREAT PRESENTATION
AND TOUR DE FORCE,
CONGRATULATIONS.
I I WANT TO TALK ABOUT WHERE THE
COLLABORATIVE DISCUSSION WITH
ARPA-H WHERE THEY'RE HEADING AND
THE TIME FRAME ON THAT.
>>THANK YOU FOR THAT.
IT'S STILL NEUTRAL -- EARLY
DAYS.
THEY HAVE ONE ON OSTEOARTHRITIS
AND ONE ON CANCER.
SO I THINK THEY'LL BE FOCUSSING
SOME OF THEIR EFFORTS LARGELY ON
THAT AND SOME OF THE DISCUSSIONS
WE'VE HAD WITH THEM ARE AROUND A
VARIETY OF OTHER ACTIVITIES AS
WELL BUT WHERE WE MIGHT BE ABLE
TO CONTRIBUTE TO THAT WORK THERE
MIGHT BE OPPORTUNITIES WITHIN
THE REAL WORLD DATA ASPECTS THAT
WE HAVE.
THOSE ARE THE KINDS OF
DISCUSSIONS WE DON'T HAVE
ANYTHING SET IN STONE JUST YET
BUT HAVING THOSE DISCUSSIONS AND
CONTINUING THAT ENGAGEMENT HAS
BEEN VERY PRODUCTIVE AND
COLLABORATIVE AND SO I THINK
THIS WILL MOVE FORWARD.
HOPEFULLY WE'LL HAVE MORE TO
TALK ABOUT AS IT CONTINUES TO
GROW AND THEY GET MORE PROGRAM
MANAGERS AS WELL IN THEIR SPACE.
AS YOU PROBABLY KNOW THEY HAVE A
LITTLE BIT DIFFERENT WAY OF
APPROACHING THE WORK THEY DO.
IT'S PROGRAM MANAGER DRIVEN.
THEY BRING PROGRAM MANGERS IN
AND THAT'S WHEN THEY START TO
PITCH IDEAS AND DISCUSS WHAT THE
IDEAS MIGHT BE.
THEN THE PROGRAM MANAGER IS
GIVEN HE FUND -- THE FUNDING
AND REASONABLE FOR DELIVERING ON
ITS ACTIVITY.
GIVEN IT'S A BOTTOM-UP APPROACH
BUT THE DISCUSSIONS GIVE A GOOD
SENSE OF CONFIDENCE IT WILL BE
PRODUCTIVE.
>>I THOUGHT IT WOULD BE A GREAT
IDEA TO INVITE THEM OVER TO SEE
THE INTRAMURAL RESOURCES.
>>WE DID TOO.
SCIENTIFICALLY SPEAKING THEY
HAVE A LOT OF INTEREST IN THE
WORK WE'RE DOING AND LARGELY
EXPERTISE IN THE GENE THERAPY
SPACE TOO.
I'M EXCITED ABOUT SOME OF THOSE
IDEAS AND OF COURSE WITH NIH AND
THEM TOO.
IT WON'T BE THE FIRST TIME WE'LL
KEEP INVITING THEM BACK TOO.
SERGIO.
>>THANK YOU FOR THE OVERVIEW.
YOU PRESENTED A LOT OF
INFORMATION.
VERY HELPFUL SEE ALL THE THINGS
BEING DONE AT NCATS UNDER YOUR
LEADERSHIP.
AS YOU WORK ON THE STRATEGIC
PLAN YOUR EFFORTS TO HAVE
LISTENING SESSIONS ACROSS THE
NATION TO DO THE ROUNDTABLE
TAKES TIME AND ENERGY FROM YOU
AND YOUR TEAM.
YOU'RE DOING IT.
THOSE ARE FANTASTIC.
I PARTICIPATE D IN ONE.
THE OTHER THING YOU'RE DOING I
WANTED TO EMPHASIZE IS REACHING
OUT TO COMMUNITY, ADVISORY BOARD
MEMBERS OF CTSAs TO LISTEN FROM
THEM AS WELL.
THEY'RE IN GOOD POSITION TO GIVE
MEANINGFUL FEEDBACK.
AND A GREAT POSITION TO PROVIDE
INPUT THAT CAN BE HELPFUL FOR
THE STRATEGIC PLANNING AND ONE
LAST THING.
I HOPE YOU RECOVER.
I'M SO SORRY MY FIRST MEET I
MISSED YOU.
I WAS LOOKING FORWARD TO SEEING
YOU AGAIN.
PLEASE BE WELL.
>>THANK YOU, SURGE YO.
AND I APPRECIATE YOUR COMMENTS
RELATED TO THE ROUNDTABLE.
WHEN WE EMBARKED ON THE
STRATEGIC PLANNING PROCESS I
WASN'T SURE HOW TO PROCEED BUT
IT MADE A LOT OF SENSE TO ME AND
TO OUR TEAM AND THEY EMBRACED IT
AS WELL.
CREDIT TO THEM BECAUSE IT'S
QUITE TIME CONSUMING.
I DID -- WE HAVE DONE 30
ROUNDTABLES SO FAR.
I'VE LOST MY VOICE.
IT HAS BEEN HELPFUL MEET THE
COMMUNITY AND STAKEHOLDERS.
WE HAVE NOT MET WILLING THE
COMMUNITY ADVISORY BOARD YET.
WE HAVE TWO MEETINGS COMING UP
LATER IN JUNE.
BUT THOSE ARE SCHEDULED AND I'M
LOOKING FORWARD TO THOSE.
WE HAD A COUPLE PUBLIC
ROUNDTABLES AS WELL AND ANOTHER
GROUP AND I REALLY APPRECIATE
THE COMMUNITY ADVISORY BOARD.
THE OTHER GROUP IS THE PATIENT
ADVISORY GROUPS PART OF THE RARE
DISEASE CLINICAL RESEARCH
NETWORK.
HEARING FROM THE PATIENT
ADVISORY GROUPS AND ADVISORY
GROUPS HAS BEEN HELPFUL AND
THERE ARE A LOT OF THEM AND HOW
YOU CAN LISTEN TO VOICES IN A
FORMAT LIKE THIS IS -- I WANTED
TO TRY TO MAKE IT AS ENGAGING AS
POSSIBLE AND ASK QUESTIONS IN
WAYS WE CAN GET WHAT'S IMPORTANT
TO THEM AND HELP US TO
PRIORITIZE THAT INPUT.
I THANK YOU FOR COMMENTING ON
THAT AND I LOOK FORWARD TO MORE
TO COME.
I ALSO THINK IT'S REALLY
IMPORTANT THIS ISN'T JUST
BECAUSE WE'RE DOING THE
STRATEGIC PLAN PROCESS BUT
SOMETHING THAT WE NEED TO
CONTINUE TO DO.
WE PUT PATIENTS AND COMMUNITIES
FRONT AND CENTER IN THE WORK WE
DO AND I THINK THAT'S GOING TO
BE IMPORTANT TO CONTINUE AS
WELL.
THAT'S GOING TO MAKE THIS A
SUCCESS NO MATTER WHAT HAPPENS
IN TERMS OF MEETING OUR ACTUAL
GOALS BUT I THINK THAT'S GOING
TO MAKE IT A SUCCESS.
I APPRECIATE THE INPUT.
PAULA, PLEASE, NICE TO SEE YOU.
>>NICE TO SEE YOU, JONI.
YOU PRESENTED A LOT OF AMAZING
PROJECTS NCATS WAS DOING.
I WAS PARTICULARLY STRUCK BY
SOME OF THE PIPELINE OR PATHWAY
PROGRAMS GOING INTO THE K-5.
WHEN YOU LOOK AT TRYING TO GET
RURAL KIDS IN UNDER SERVED
POPULATIONS AND DISADVANTAGED
POPULATIONS INTO SCIENCE OR
MEDICINE OR HEALTH CARE, THEY
FALL BEHIND IN MATH AND SCIENCE
AND THEN IT DOESN'T MATTER IF
YOU HIT THEM IN HIGH SCHOOL
THEY'RE ALREADY TOO FAR BEHIND.
I'M WONDERING IF YOU CAN SHARE
SOME OF THE THINGS NOT HERE BUT
WE HAVE A LOT OF RURAL PROGRAMS
AT TEXAS A&M AND IT SEEMS LIKE
THE ROLE PLAYS FOR THE K-5 WOULD
FIT IN WELL WITH WHAT WE'RE
TRYING TO DO AND I'M SURE WHAT
OTHERS ACROSS THE COUNTRY ARE
TRYING TO DO.
>>YEAH, THANK YOU FOR THAT
COMMENT.
THAT'S A GREAT IDEA.
MAYBE IT COULD BE ENCOURAGING
FOR CTSA PROGRAMS AND ENGAGEMENT
IN OUTREACH AND THAT WOULD BE
TERRIFIC.
I'D LOVE TO TALK TO YOU MORE
ABOUT SOME OF THOSE IDEAS.
WE'RE HAPPY TO SHARE AND FOLLOW
UP DIRECTLY ON THAT AND PERHAPS
GIVE YOU SOME OF THE WORK WE'VE
BEEN DOING.
ANNA IS TELLING ME WE'RE A
LITTLE BIT OVER TIME SO I'M
GOING TO JUST MOVE US ALONG HERE
AND REMINDED WE HAVE A BIT OF
TIME AT THE END OF THE DAY IN
ADDITION TO THE OTHER
CONVERSATIONS AND DISCUSSIONS
WE'LL HAVE THROUGHOUT THE
REMAINDER OF THE AGENDA BUT BUT
I THINK WE CAN GO AHEAD AND MOVE
ALO
ALONG.
WE'RE GOING IN THE NEXT SESSION
ON THE STRATEGIC PLAN
DISCUSSION.
I'D LIKE TO DEUCE MEREDITH
TEMPLE O'CONNOR AND PEN YNY
BERGUNE.
PENNY IS THE DIRECTOR OF THE
OFFICE AND MEREDITH IS THE
SCIENCE POLICY DIRECTOR AS WELL
AND THEY'RE USHER THE NEXT
SESSION.
>>THANK YOU, EVERYBODY.
I'M HAPPY TO GIVE THE COUNCIL
ALONG WITH DR. O'CONNOR WHERE WE
ARE IN THE NCATS 2024 STRATEGIC
PLANNING PROCESS.
OUR CURRENT NCATS STRATEGIC PLAN
IS DATED FALL OF 2016.
IT'S WELL OVERDUE TO DO A DEEP
DIVE INTO ENGAGING OUR
COMMUNITIES WHAT WE SHOULD BE
THINKING ABOUT AND THE GAPS AND
OPPORTUNITIES TO ADDRESS.
WITH THAT I'M GOING TO HAVE
MEREDITH LEAD THE PRESENTATION
AND I'LL PICK UP ON THE BACK
END.
GO AHEAD, MEREDITH.
>>THANK YOU SO MUCH.
CAN YOU ALL HEAR ME OKAY?
GREAT.
WE'RE THINKING ABOUT A ROAD MAP
FOR THE DECADE AHEAD.
.
TYPICALLY STRATEGIC PLANS ARE
ABOUT FIVE YEARS.
OURS IS A LITTLE BIT OLDER AND
THERE'S REASONS FOR THAT I'LL
DIVE INTO IN A SECOND BUT WE'RE
DELIGHTED TO HAVE KICKED THIS
OFF AND BE ENGAGING ACROSS THE
BOARD WITH DIFFERENT
STAKEHOLDERS AND IN DIFFERENT
WAYS AND FORMATS FOR THIS
PROCESS.
AS DR. RUTTER TALKED ABOUT A LOT
AND IN INTERNAL ENGAGEMENT
SESSIONS AND JONI, DON'T MEAN TO
SPEAK FOR YOU SO FEEL FREE TO
CHIME IN BUT IT'S ABOUT
CO-DESIGNING A STRATEGIC PLAN.
THIS IS SOMETHING WHERE WE DO
VERY MUCH VALUE THE INPUT FROM
OUR DIFFERENT STAKEHOLDERS AND
WANT TO THINK OF THIS AS AN
ACTIONABLE ROAD MAP TO ACHIEVING
THEACIOUS --
THE AUDACIOUS GOALS.
THE TIME LINE WE STARTED TO GET
ORGANIZED AS SOON AS SHE WAS
NAMED DIRECTOR AND WE START THE
STRATEGIC PLAN BUT WHEN
DR. AUSTIN ANNOUNCED HIS
DEPARTURE DR. RUTTER DECIDED IT
WAS BEST TO WAIT UNTIL A
PERMANENT DIRECTOR WAS NAMED AND
AS SOON AS SHE WAS NAMED WE
STARTED MOVING FORWARD AGAIN ON
THE PLAN.
THIS HOPEFULLY GIVES A SENSE OF
THE TIME LINE WE'RE WORKING WITH
AND SOME OF THE ACTIVITIES WE'RE
UNDERTAKING AS PART OF THE
STRATEGIC PLANNING PROCESS.
WE ARE HOPING AND TARGETING FOR
A ROLLOUT IN EARLY 2024 BUT WE
ALSO JUST NOW WITH THE PUBLIC
ROUND MEETINGS WE'RE WRESTLING
WITH DATA WE WANT TO CAREFULLY
AND DELIBERATELY WORK THROUGH.
THIS THE TARGET DATE BUT DON'T
HOLD ME TOO IT IF IT TAKES
LONGER TO GET THROUGH THINGS.
WE WANT TO BE THOUGHTFUL ABOUT
THE INPUT WE'RE GETTING AND HOW
IT SHAPES OUR STRATEGIC PLAN.
WE'RE VERY MUCH IN THE INITIAL
STAKEHOLDER ENGAGEMENT PART AS
DR. RUTTER TALKED ABOUT AND HAD
ROUNDTABLE INTERNALLY AND
EXTERNALLY.
SHE LAID OUT A PRESENTATION WITH
HER VISION AND AUDACIOUS GOALS
AND WE WALKED THROUGH A SET OF
QUESTIONS.
WE'LL CONTINUE WITH A MORE
OPEN-ENDED DISCUSSION WITH THE
ADVISORY COUNCIL AND DR. RUTTER
AND TWO OTHERS.
AFTER WE START TO SYNTHESIZE THE
FEEDBACK WE'RE PLANNING TO PUT
OUT REQUEST FOR INFORMATION.
ONE FOR THE PUBLIC AS
STAKEHOLDERS ON THE PUBLIC SIDE
AS WELL AS FOR OUR STAFF.
WE'D LIKE TO DIGEST THE INPUT
RECEIVED BEFORE WE START TO
SHAPE THAT.
THE OTHER THING IS WE WANT THIS
TO BE ROBUST ALIGNING WITH OUR
MISSION AND VISION ECHOING OUR
VALUES AND SHARED VALUES WITH
OUR COMMUNITY.
AS MENTIONED AND SHE'S MENTION
ED AND WHILE HER GOALS ARE
THOUGHT TO BE INSPIRATIONAL AND
WE WANT TO MAKE SURE IN THE
INTERVENING YEARS THE
ACTIVITIES, GOALS, OBJECTIVES WE
LAY OUT ARE OBTAINABLE AND
MEASURABLE.
SOMETHING ELSE I'LL SHARE IS A
CONVERSATION DR. RUTTER AND
OTHERS INTERNALLY HAVE HAD IS
THIS IS NOT SOMETHING WE WANT TO
PUT ON THE SHELF FOR FIVE YEARS.
THEN SAY HOW DID WE DO?
THIS IS SOMETHING WE WANT TO
THINK ABOUT MAYBE NOT QUITE REAL
TIME BUT CLOSER TO THAT.
CHECKING IN WITH OURSELVES AND
STAKEHOLDERS AFTER THE PLAN IS
RELEASED TO TAKE WITH OURSELVES
WHERE WE ARE.
WE'RE AN AGILE ORGANIZATION THAT
MOVES TOWARDS OPPORTUNITIES AND
IT'S A BIG ROBUST SPACE AND WE
WANT TO MAKE SURE OUR PLAN IS IN
THAT FRAME AS WELL.
IT'S ALSO A POWERFUL TOOL TO BE
SUCCESSFUL AND CHECKING IN WITH
OURSELVES ON A ROUTINE BASIS.
AS DR. RUTTER LIKES TO TALK
ABOUT A LOT WE'RE OKAY AND
COMFORTABLE WITH RISK AND
FAILURE BUT WANT TO FAIL FORWARD
AND LEARN AND MOVE FORWARD FROM
IT.
THIS SHOULD BE FAMILIAR TO MOST
STAKEHOLDERS AND THE PUBLIC
JOINING ON THE VIDEOCAST AND THE
ADVISORY COUNCIL, THIS IS
DR. RUTTER'S VISION OF MORE
TREATMENTS FOR ALL PEOPLE MORE
QUICKLY.
AND THIS WILL LIKELY INFORM THE
STRATEGIC PLAN.
THESE ARE THE THREE AUDICIOUS
GOALS IN CASE YOU HAVEN'T HAD AN
TONIGHT TO HAVE HEARD ABOUT
THEM.
BY THE WAY, I'LL ALSO MAKE A
SMALL SIDE NOTE WE'LL BE POSTING
THE PUBLIC ROUNDTABLE RECORDING
DR. RUTTER PRESENTED AT
PUBLICLY.
IF FOLKS HAVEN'T HAD THE
OPPORTUNITY TO HEAR THESE PIECES
WE'LLING POSTING THAT AND WE'LL
HAVE A MAILBOX THEY ARE
AUDACIOUS.
IT'S EXCITING TIME TO BE WORKING
AT NCATS AND SEE US TRY TO
HARNESS THE POTENTIAL THAT IS
THERE IN THE TRANSLATIONAL
SCIENCE SPACE.
THEY'RE QUITE ASPIRATIONAL AND
SOMETHING WE WANT TO BUILD TO.
I WILL SAY THEY'RE NOT THE SAME
AS THE GOALS OF THE STRATEGIC
PLAN.
WE HAVE NOT PUT PEN TO PAPER ON
THE STRATEGIC PLAN BECAUSE THE
INPUT PIECE IS IMPORTANT TO OUR
WORK TO MAKE SURE WE GET THAT
AND HAVE AN OPPORTUNITY TO
DIGEST IT BEFORE WE START TO
WRITE THE PLAN.
I WANT TO BE CLEAR BECAUSE IT'S
BEEN CONFUSING FOR FOLKS AT
TIMES DURING DURING SOME
FEEDBACK SESSIONS TO MAKE SURE
IT'S CLEAR THESE ARE NOT THE
SAME AS WHAT WE'LL PUT TOGETHER
FOR THE STRATEGIC PLAN.
WILL SET HOW WE'RE THINKING
ABOUT THE STRATEGIC PLAN.
THE ALL TREATMENTS FOR ALL
PEOPLE IS MORE FEEDBACK AND THE
MEETINGS AS WELL.
IT'S NOT ALL WE DO AND THINK
ABOUT.
THESE ARE OBVIOUSLY SUPER
IMPORTANT ASPECTS OF WHO WE ARE
AS A CENTER AND WHAT WE THINK
ABOUT.
WE HAVE OTHER AREAS ON OUR RADAR
FOR STRATEGIC PLANNING.
I HAD THE OPPORTUNITY TO BE PART
OF THE NIH STRATEGIC PLAN AND
THEY TALKED ABOUT CROSS-CUTTING
THEMES.
WE'VE TALKED ABOUT THESE IDEAS
IN SOME SOMEWHAT THEY DON'T KNOW
QUITE YET HOW WE'LL SHAPE THEM
UP AND FIGURE THEM INTO THE
STRATEGIC PLAN BUT THAT'S ONE
WAY WE'RE THINKING ABOUT THEM.
COULD THEY BE CROSS-CUTTING
THEMES OR SPECIFIC TOPIC AREAS
WITHIN OUR GOALS AND OBJECTIVES
BUT I JUST WANTED TO MAKE SURE
FOLKS ARE CLEAR THAT IN ADDITION
TO THE MORE TREATMENTS FOR ALL
PEOPLE, MORE QUICKLY AND THE
AUDACIOUS GOALS WE'RE THINKING
ABOUT OTHER PIECES AND HOW THEY
FIT INTO THE BROADER CONTEXT AND
FRAMING OF WHO WE ARE.
THE ONE THING I'LL MENTIONED.
MOST WOULD BE VERY FAMILIAR TO
OUR STAKEHOLDERS AND ADVISORY
COUNCIL.
THERE'S A LOT OF THINGS WE
ALREADY DO ON A REGULAR BASIS
BUT JUST WANT TO MAKE SURE FOLKS
REALIZE WE'RE THINKING ABOUT
THESE AS WELL.
STEWARDSHIP IS ONE OF THE
REQUIREMENTS IN THE NIH CENTERED
STRATEGIC PLAN.
THE REST ARE OUR THINKING ABOUT
DIFFERENT AREAS WE WANT TO HAVE
ON THE RADAR FOR OUR STRATEGIC
PLAN.
THIS IS WHERE I'M GOING HAND OFF
TO DR. BERGOON IN THE ROOM.
I TO BE THERE IN PERSON BUT
SHE'S GOING TO WALK YOU THROUGH
SOME ACTIVITIES.
OBVIOUSLY YOU ALREADY SAW THIS
IN THE WEALTH OF WAYS WE'RE
TRYING TO ENGAGE WITH
STAKEHOLDERS.
SHE'S GOING TO WALK YOU THROUGH
SOME OF THE WORK WITH THE PUBLIC
ROUND TABLES AND INITIAL
FEEDBACK AND WE'LL WORK WITH
DR. RUTTER TO OPEN IT UP FOR A
COUNCIL DISCUSSION AND I LOOK
FORWARD TO BEING PART OF THAT AS
WELL.
>>THANK YOU.
WE GATHERED INPUT FROM MANY
STAKEHOLDERS BOTH INTERNALLY AND
EXTERNALLY ABOUT WHERE WE SHOULD
GO AND THINK ABOUT IN OUR NEXT
STRATEGIC PLAN.
THIS IS COLOR CODED.
AND THEY HAVE WORKED WITH THE
CLINICAL AND TRANSLATIONAL
SCIENCE PROGRAMS P.I.s AND THEIR
STAFF ABOUT DIFFERENT WAYS THEY
CAN ENGAGE AN PROVIDE INPUT IN
THE PLANNING EFFORT.
ALSO ENGAGED WITH THE RARE
DISEASES COMMUNITY TALKING TO
BOTH THE RDCRN AND THE PATIENT
ADVOCACY GROUPS ON THE STRATEGIC
PLANS.
THE PUBLIC ROUNDTABLES WERE TWO
WEEKS AGO WERE VERY GOOD
WELL-ATTENDED MEETINGS.
I'LL GIVE A BIT OF THE DETAILS
IN THE NEXT SLIDES.
AND TO GET FEEDBACK AND
REFLECTIONS IF YOU ATTENDED THE
ROUNDTABLES THEMSELVES.
AND WE'RE NOT DONE YET.
WE HAVE A FEW MEETINGS IN JUNE
AND THERE'S ADDITIONAL MEETINGS
THAT ARE GOING TO POP UP ON THE
SCHEDULING RADAR BECAUSE THERE'S
OTHER GROUPS AND WHERE NCATS CAN
BE GOING OVER THE NEXT FIVE TO
10 YEARS.
WE HAD THREE GENERAL QUESTIONS.
WHAT ARE THE BIGGEST
OPPORTUNITIES FOR NCATS TO
ENGAGE AND TO INVEST OVER THE
NEXT FIVE TO 10 YEARS.
THE OTHER ONE WAS WHAT ARE THE
BIG GAPS AND OPPORTUNITIES WE
SHOULD BE THINKING ABOUT AND THE
LAST ONE IS WHAT'S MISSING?
IS THERE ANYTHING ELSE WE SHOULD
THINK ABOUT AS WE MOVE FORWARD
IN TRYING TO ADVANCE
TRANSLATIONAL SCIENCE?
AS MANY OF YOU KNOW IF YOU
PARTICIPATED IN OUR ROUNDTABLES,
WE HAD THREE QUESTIONS WE BOOKED
OUT INTO SEPARATE BREAKOUT
DISCUSSIONS SO WE COULD GET
DIFFERENT PERSPECTIVES ON THOSE
SAME QUESTIONS.
THE BREAKOUT SESSIONS WERE FROM
THE RESEARCH PERSPECTIVE,
DIVERSITY, EQUITY, INCLUSION AND
ACCESSIBILITY PERSPECTIVE.
COMMUNITY PATIENT ENGAGEMENT
PERSPECTIVE AND TRAINING AND
EDUCATION.
WE HAD A GOOD TURNOUT OF OVER
200 PARTICIPANTS OVER 300
REGISTRANTS.
AND PUBLIC CITIZENS AND
GOVERNMENT SECTORS.
NCATS THE FIRST PLAN WAS DATED
AND THIS IS WHAT WILL SHOW YOU
AN ICE BREAKER AT THE ROUNDTABLE
AND WHEN WE HAVE THE QUESTION
AND THINK OF NCATS WHAT WORDS
COME TO MIND.
I FELT LIKE WE WERE SEEN WITH
THESE WORDS AND THINGS NCATS
WORK WITH YOU ON AND VIEW
OURSELVES AS IMPORTANT TO HOW WE
GET TRANSLATIONS DONE
EFFICIENTLY AND EFFECTIVELY.
HERE'S WHAT HAPPENED AT THE
ROUNDTABLE.
IF YOU TOOK ONE QUESTION THE
UNMET NEEDS OR GAPS IN
TRANSLATION, THAT SHOULD BE
ADDRESSED BY NCATS.
THIS IS PRETTY SMALL PRINT BUT
FROM THE RESEARCH PERSPECTIVE
WARE GETTING RESPONSES SUCH AS
WE NEED TO THINK ABOUT
REPRESENTATIVE IN CLINICAL TRIAL
OR NEED HUMAN-BASED BIOLOGY
BASED RESEARCH HUBS AT
INSTITUTIONS AROUND THE COUNTRY.
WE THOUGHT ABOUT THIS FROM THE
COMMUNITY ENGAGEMENT
PERSPECTIVE.
WE GOT COMMENTS SUCH AS TRIAL
READINESS FOR MANY RARE DISEASES
IS LACKING EVEN IF THERE'S A
PROMISING THERAPY WE DON'T KNOW
HOW TO DESIGN A TRIAL.
FROM THE TRAINING AND EDUCATION
PERSPECTIVE THERE'S GAPS OR
UNMET NEEDS WERE FOCUSSED ON THE
JOURNEY FROM DISCOVERY TO HEALTH
SOLUTIONS.
THE FIRST IN HUMAN STUDIES AND
REGULATORY SCIENCE,
COMMERCIALIZATION.
HOW DO WE LEARN ABOUT THAT?
THAT'S A GAP IN OPPORTUNITY FROM
THAT SPACE.
FROM DIVERSITY, EQUITY,
INCLUSION AND ACCESSIBILITY
SPACE, THERE WAS A LOT OF
DISCUSSIONS ABOUT ACCESS.
HOW DO WE GET NEW TREATMENTS TO
THE UNDER SERVED COMMUNITIES.
THIS IS A REFLECTION OF HOW WE
COULD ASK THE SAME QUESTION AND
GET A MORE HOLISTIC RESPONSE AND
IMPRESSIONS FROM THE COMMUNITY
AND PUBLIC WHAT WE COULD BE
THINKING ABOUT.
SO YOU DON'T NEED TO REVIEW ALL
THESE BUT THESE THE TAKEAWAYS
FROM THE BREAKOUT DISCUSSIONS
ACCORDING TO THE FOUR SEPARATE
BREAKOUT ROOMS.
WE SEARCHED THROUGH COMMENTS
ABOUT DISSEMINATION AND
IMPLEMENTATION AND COMMENTS ON
PREVENTION.
I WILL TELL YOU THAT THOSE TWO
THINGS WERE COMMON TO THE PUBLIC
ROUNDTABLES AND HEARD MULTIPLE
TIMES IN THE INTERNAL
DISCUSSIONS WITH OUR STAFF.
THERE WERE COMMENTS AROUND DATA
SHARING AS WELL AND YOU CAN READ
THEM ON YOUR OWN.
WE'LL MAKE THIS VIDEO OR WEBINAR
AVAILABLE LATER FOR SUBSEQUENT
VIEWING.
IN DIVERSITY, EQUITY, INCLUSION
AND ACCESSIBILITY THERE WERE
MANY COMMENTS ABOUT BUILDING
TRUST.
THERE'S ALSO A NEED TO
UNDERSTAND WHAT TRANSLATIONAL
I
SCIENCE IS AND WHAT IT DOES AND
COMMENTS ABOUT BUILDING BRIDGES
WITH NEW PARTNERS TO MAKE THAT
HAPPEN.
BREAKOUT DISCUSSIONS.
SEVERAL COMMENTS ABOUT PATIENTS
AS PARTNERS AND THEY NEED TO BE
ENGAGED EARLY IN THE PROCESS.
WE NEED TO HAVE THE COMMUNITY AS
PART OF THE RESEARCH DESIGN AND
TO ADDRESS ISSUES OF ACCESS AND
ADOPTION.
TRAINING AND EDUCATION
PERSPECTIVES.
A FOCUS ON TEAM SCIENCE AND
TRANSLATIONAL SCIENCE TRAINING
AND WORKFORCE DIVERSITY.
THE EDUCATION SPACE THERE WERE
SEVERAL COMMENTS ABOUT CAREER
TRANSLATION.
WE'RE ALSO WORKING WITH PEOPLE
AS THEY TRY TO MOVE THINGS AND
ADVANCE THEM TOWARD HOW DO YOU
THE UNDERSTANDING OF WHAT THE
TRANSITIONS ARE AND SOME PEOPLE
WANT TO USE THEM AS A CAREER
PATHWAY.
THESE WERE SOME OF THE THINGS WE
SAW IN THE CROSS-CUTTING AREAS.
AS I SAID BEFORE, MANY COMMENTS
ABOUT PREVENTION.
THERE'S SOME VERY INTERESTING
ONES ABOUT DON'T JUST MAKE MORE
TREATMENTS, MAKE BETTER
TREATMENTS.
LET'S FOCUS ON THE CAUSES OF
THOSE DISEASES NOT JUST THE
TREATMENTS.
AGAIN, DISSEMINATION AND
IMPLEMENTATION WAS A FREQUENT
COMMENT.
THERE WERE SEVERAL COMMENTS
ABOUT NCATS CAN DO SOMETHING
ABOUT NOVEL CLINICAL TRIAL
APPROACHES AND HOW DO WE MAKE
THAT MORE BROADLY ACCESSIBLE AND
DISSEMINATE THAT.
HEALTH EQUITY HAS BEEN A HUGE
DISCUSSION NOT ONLY IN THE
PUBLIC REALM BUT ALSO IN THE
INTERNAL DISCUSSION AND MORE
PARTNERSHIPS AND COLLABORATIONS
AND PATIENTS HAVE TO BE AN
IMPORTANT PART OF THAT TEAM.
THERE WERE COMMENTS ABOUT MAKING
BRIDGES AND HELPING WITH THE
ADVANCEMENT TO THE NEXT SECTOR
WHETHER DECISION MAKE ORG
BUSINESS OR WHAT HAVE YOU.
WE WANT TO REDUCE ADMINISTRATIVE
BURDEN AND HELP IN RESEARCH AND
CARE AND HELP FOSTER A
TRANSLATIONAL SCIENCE WORKFORCE.
BEFORE I HAND THIS OVER TO JONI
FOR DISCUSSION, I WANTED TO
ACKNOWLEDGE THE TEAM HERE THAT
HAS TAKEN US TO THIS POINT IN
THE PROCESS AND STILL HAVE MORE
TO GO.
WE HAVE OUR STRATEGIC PLANNING
TEAM WHICH HAS BEEN FOCUSSED ON
GETTING THIS OFF THE GROUND AND
WE'LL WORK ON THE DRAFT.
COMMUNICATIONS, I.T. AND WORK
TEAMS HAVE HELPED DISSEMINATION
OF THE INFORMATION AND OUTREACH
TO THE PUBLIC AND STAKEHOLDERS.
AND IN THE ROUNDTABLE I WANTED
TO THANK THE NCATS LEADERSHIP
THAT HELPED WITH THE BREAKOUT
DISCUSSIONS.
I THINK THEY WERE RICH IN
DISCUSSION AND HAVE BEEN
BENEFICIAL IN MOVING FORWARD.
AND WITH THAT I'M GOING TO HAND
THIS TO YOU IN TERMS OF HOW WE
CAN HAVE A COUNCIL DISCUSSION
AND THEN MOVE FORWARD.
>>THANK YOU VERY MUCH.
AND WE HAVE A VARIETY OF NCATS
STAFF THAT ARE LISTENING IN AND
HAVE BEEN A PART OF THE
PROCESSES AND MAY ENGAGE AS
WELL.
AND I WANT TO TALK ABOUT THE
PROCESS AND PARTICIPATION FROM
THE COUNCIL MEMBERS IN OUR OWN
LITTLE ROUNDTABLE IF YOU WILL.
LARGELY I'M INTERESTED AND I
KNOW SOME HAVE BEEN A PART OF
THE ROUNDTABLE DISCUSSIONS AND
SER
SERGIO, THANK YOU FOR YOUR
COMMENTS ON THE IMPORTANCE AND
THINKING ABOUT THE COMMUNITY
ENGAGEMENT THAT'S HAPPENING.
I'M ALSO WONDERING FROM YOU ALL
THAT FROM THOSE ABLE TO
PARTICIPATE WHAT HAS STUCK OUT
TO YOU IN TERMS OF THE
ROUNDTABLE DISCUSSIONS?
AND EVEN IN THESE FOUR AREAS
THAT WAS MENTIONED WITH
DIVERSITY, EQUITY, INCLUSION AND
ACCESSIBILITY AND RESEARCH AND
COMMUNITY ENGAGEMENT AND PATIENT
AND TRAINING AND ENGAGEMENT.
THESE ARE KEY THEMES THE BASIS
OF WHERE WE CAN EXPLORE
DEVELOPING A STRATEGIC PLANNING.
THESE IDEA ARE MEANT TO CUT
ACROSS ALL THESE DIFFERENT AREAS
AND WHERE CAN WE PROVIDE THAT
PRIORITIZATION AND INPUT FROM
THE THINGS THAT HAVE JUMPED OUT
AT YOU DURING THE ROUNDTABLE
DISCUSSIONS?
I'D LIKE TO HEAR WHAT YOUR
COMMENTS AND INPUT WOULD BE FROM
YOUR OWN PARTICIPATION AND THEN
I'D LOVE TO HEAR -- WE'LL GO
INTO OTHER QUESTIONS THAT I'LL
ASK AROUND THE STRATEGIC
PLANNING PROCESS AND SOME
QUESTIONS I'D LIKE TO ASK YOU.
WOULD ANYBODY WANT TO JUMP IN
AND PROVIDE COMMENTS AROUND THE
ROUNDTABLE TABLES?
>>I HAD A CHANCE TO ATTEND THE
ROUNDTABLE.
I THOUGHT THE WAY IT WAS
STRUCTURED WAS VERY INNOVATIVE.
YOU ASK THE QUESTION WITH
DIFFERENT GROUPS THAT HAD
DIFFERENT PERSPECTIVES AND WHAT
WAS INTERESTING WAS SEEING HOW
THAT WERE THEMES THAT CAME OUT
AND TIED TOGETHER AND SHOWED HOW
AS EXPECTED DIFFERENT GROUPS TIE
TOGETHER A SYSTEMS APPROACH TO
ACHIEVE THE GOALS.
FOR EXAMPLE TRYING TO BUILD OUT
AND EXPAND THE TRANSLATIONAL
SCIENCE WORKFORCE IS ALL THAT
MATTERS IN TRYING TO GET MORE
TREATMENT MORE QUICKLY.
THERE'S JOB DESCRIPTIONS WE
PROBABLY DON'T EVEN KNOW OR
THINK ABOUT YET THAT NEED TO BE
THERE PARTICULARLY IN INDUSTRY
IF WE WANT TO TAKE THIS FAR.
SEGUE INTO THAT ONE OF THE
SUGGESTIONS MADE MAYBE HAVING AN
INDUSTRY-SPECIFIC ROUNDTABLE AS
SHOWN IN THE NUMBERS.
THERE WASN'T A HUGE
PARTICIPATION FROM INDUSTRY BUT
HAVING THEIR INPUT FOR THE GOAL
YOU'RE TRYING TO ACHIEVE IS KEY
AND ALSO AN OPPORTUNITY TO GET
MORE TO INDUSTRY AND NCATS AS
YOU'RE DOING HERE.
ANOTHER THING THAT STUCK OUT
THAT CAME UP A COUPLE TIMES WAS
COST WHICH IS PROBABLY NOT IN
YOUR WHEELHOUSE BUT THERE WAS A
TIME WHERE WE CAN GET THINGS
MORE EFFICIENTLY OUT THE COST
WOULD DROP.
AT LEAST RECOGNIZING THE COST TO
HEALTH EQUITY AND ACCESS IS A
GOAL.
PARTICULARLY WITH SELLING GENE
THERAPY WE KNOW IS EXPENSIVE.
>>THANK YOU FOR THE COMMEN
COMMENTS,ANIKA.
>>I LOVE THE IDEA OF A MORE
SPECIFIC WAY OF ENGAGING
INDUSTRY AND DOING A ROUNDTABLE.
THAT'S A GREAT IDEA AND WE CAN
CERTAINLY INCORPORATE THAT TOO.
AND I THINK IT'S IMPORTANT TOO
BECAUSE WHEN YOU MENTIONED THE
IDEA OF THE WORKFORCE AND
THINKING ABOUT ALL THESE JOBS
OUT THERE THAT WE DIDN'T EVEN
KNOW ABOUT YET BUT WE NEED TO
BUILD A DIFFERENT WORKFORCE FOR
WHERE WE'RE GOING BIOMEDICAL
SCIENCES AND THINKING FOR
EXAMPLE SOME OF THE WORK WE'RE
DOING IN THE WINDOW THAT I
TALKED SOMEWHAT ABOUT TODAY IN
TERMS OF GENE THERAPY AND
DEVELOPMENT OF GENE THERAPY
PROGRAMS, THE DELIVERY, THE
OTHER NON-VIRAL KIND OF DELIVERY
AND THE WORK THAT NEEDS TO GO ON
IN THAT SPACE AND TRANSLATING
THAT TO THE CLINICAL SPACE AND
THINKING ABOUT THE MANUFACTURING
WORK AND CLINICAL TRIAL WORK AND
QUALITY WORK AND DRUG
DEVELOPMENT WORK.
THESE ARE FAIRLY NASCENT FIELDS
WHERE I THINK WE REALLY DO NEED
TO THINK ABOUT WHAT'S THE
WORKFORCE NEEDED TO DO THAT AND
THAT WORKFORCE NEEDS TO BE AT
EVERY LEVEL FOR SURE.
I LIKE THE IDEA OF TRAYING TO
-- TRYING TO MAP THAT OUT AND
WORKING ONE THE WORKFORCE TO
HELP UNDERSTAND THE
POSSIBILITIES OF A CAREER IN
SCIENCE TO ADVANCE THESE
ACTIVITIES AND THE OTHER SPACE
IN THE PRECLINICAL SIZE I THINK
OF THE HUMAN-BASED CELL MODELS
WE'RE TRYING TO DEVELOP AS WELL
WE KNOW THERE'S A 90% OF FAILURE
RATE OF DRUG CANDIDATES THAT
MOVE INTO CLINICAL TRIALS AND
MOST IS IN THE CLINICAL TRIAL
SPACE AND PRE-CLINICAL AND
CLINICAL ASPECTS CONTRIBUTE AND
ON THE CLINICAL SIDE AND
UNDERSTANDING THE RODENT MODEL
HAVE BEEN THE GOLD STANDARD
FOREVER ARE REALLY NOT CUTTING
IT FOR WHAT WE NEED TO DO IN
TERMS OF DRUG DEVELOPMENT.
REENGINEERING USING DIFFERENT
DISCIPLINES AND BACKGROUNDS TO
DEVELOP THE MODELS MORE
HUMAN-CELL BASED AND FIT FOR
PURPOSE FOR THE KIND OF DRUG
DEVELOPMENT PROGRAMS WE'LL BE
DOING IS ALSO IMPORTANT IN
BRINGING IN THAT TRANS
DISCIPLINARY SCIENCE IS
SOMETHING WE CAN HELP CONTRIBUTE
FOR THAT WORKFORCE.
THESE ARE GREAT COMMENTS AND
ANIKA I KNOW THERE'S PROBABLY A
LOT OF WORK WE CAN DO ON THIS
TOGETHER SO THAT'S EXCITE.
THANK YOU.
I SEE ROBIN AND ANDY AND SURGIO.
>>AS YOU WERE TALKING IT
REMINDED ME OF ANOTHER COMMENT
THAT COULD BE HELPFUL IN THE
ATTORNEY GENERAL STRATEGY TO
HAVE A GRAPHIC OF THE
TRANSLATIONAL SCIENCE PROCESS.
I KNOW YOU HAVE THE MAP THAT MAY
BE HARDER TO DO THIS BUT SOME
TYPE OF GRAPHIC TO SHOW THE
PROCESS AND WHERE NCATS PLANS TO
CONTRIBUTE AS ARTICULATED IN THE
STRATEGY.
SO MANY PIECES YOU CAN'T DO IT
ALL BUT I THINK THE GRAPHIC MAY
HELP ARTICULATE WHERE NCATS IS
PLANNING TO CONTRIBUTE BUT THEN
SHOWS THE LINKAGE OF WHAT THAT
MEANS TO THE DOWN STREAM OR
UPSTREAM PIECES.
VISUALS ARE HELPFUL SO IT WOULD
GO A LONG WAY IN TRANSLATIONAL
RESEARCH TO GET TO THE WORK WE
CAN DO SINCE WE CAN'T DO
EVERYTHING WHERE CAN WE
PRIORITIZE AND WHERE CAN THEY BE
MORE TANGIBLE.
ROBIN AND THEN ANDY AND THEN
SERGIO.
>>I ALSO THOUGHT THIS WAS
ENJOYABLE.
I LOVE HELPING MORE PEOPLE MORE
QUICKLY BUT MY GUT SAYS IT'S
GOOD STUFF BUT THE SLOGAN
DOESN'T CAPTURE ALL THE
COMMUNICATION.
PREVENTION AND ON COVID, YOU'RE
PREVENTING DEATHS AND
HOSPITALIZATION WHICH IS GOOD
BUT I THINK MOST PEOPLE WOULD
RATHER NEVER GET THE DISEASE
THAN WORRY ABOUT WHAT'S
TREATABLE.
I THINK WE'RE MISSING ENGAGING
ACADEMIC CLINICIANS BUT NOT OUT
IN THE WORLD CLINICIANS AND
WHERE THE VAST MAJORITY OF
PEOPLE GET ACCESS TO HEALTH CARE
AND HAVE TREATMENTS FOR INTEREST
IN THE HEALTH CARE SYSTEM AND
YOU'VE GOT INDUSTRY INPUT BUT I
DON'T THINK YOU GOT INPUT FROM
THE MAJOR ORGANIZATIONS THAT WE
THINK ABOUT THAT RUN MOST OF OUR
HOSPITALS AND LARGE
CORPORATIONS.
THAT ARE THE GAY GATE KEEPERS
AND TALKING ABOUT IMPLEMENTATION
AND DISSEMINATION HOW YOU GET
SOME OF THESE TREATMENTS INTO
SETTINGS WITH HEALTH CARE.
MAYBE THINK OF ENGAGING THAT
SECTOR TOO.
>>I WANTED TO RESPOND
CONSISTENTLY IN OUR INTERNAL
ROUNDTABLE AND EXTERNAL
ROUNDTABLE.
I'D CALL IT THE FIRST MODEL/LAST
MODEL OF TRANSLATION WHERE IF WE
DEVELOP ALL THESE TREATMENTS AND
IT NEVER GETS TO THE PEOPLE AND
IF WE DEVELOP THE TREATMENTS OR
THINK ABOUT DIFFERENT WAYS TO
PREVENT OR MINIMIZE POTENTIAL
DISEASES IN THE FIRST PLACE AND
WE HAVE THE TECHNOLOGY TO DO IT
BUT WE DON'T ADDRESS IT AND SO
WE'RE HEARING THIS VERY LOUD AND
CLEAR FROM BOTH OUR INTERNAL
STAKEHOLDERS AS WELL AS OUR
EXTERNAL ONES.
I APPRECIATE THAT COMMENT AND
I'M SURE IT WILL BE SOMETHING
THAT WE WILL HOLD IN AS WE HAVE
THE COMMUNICATION.
WE THINK WE'VE GOT IT BUT IT'S
NOT GETTING OUT THERE.
>>YEAH.
AND TO THAT POINT TOO, I AGREE
WITH PENNY WE HEARD THIS A BIT
AND I AGREE WITH THIS COMMENT.
I THINK WE NEED TO FIGURE OUT
HOW WE CAN DO THIS BETTER AND
ESPECIALLY IN THE IDEA OF
DISSEMINATION AND
IMPLEMENTATION.
WE CAN DO A GOOD JOB
DISSEMINATING BY PUBLISHING
PAPERS BUT THAT'S NOT WHAT WE
MEAN AND MAKE SURE THE WORK
WE'RE DOING CAN BE ADOPTED.
HOW CAN IT BE ADOPT?
HOW CAN WE INCENTIVIZE THAT
ADOPTION IS WHAT WE WANT WITH
DISSEMINATION AND
IMPLEMENTATION.
WHEN YOU TALK ABOUT THE IDEA OF
REACHING THE CLINICIANS OUT IN
THE COMMUNITIES WHO AREN'T
REALLY ASSOCIATED WITH THE
ACADEMIC ORGANIZATIONS, WE STILL
HAVE A LOT OF WORK TO DO WITH
THE ACADEMIC COMMUNITIES.
HOW CAN WE PUSH THE ENVELOPE AND
GET IT OUT TO THE COMMUNITIES TO
ADOPT AND TALKING TO THE C SUITE
PEOPLE MAY HAVE INTERESTING
INSIGHT AND THAT'S A GREAT IDEA.
THANK YOU.
THE DEI PIECE IS REALLY
IMPORTANT.
ANNIE.
>>I THINK THE ROUNDTABLE TABLES
HAVE BEEN PHENOMENAL AND THEY'RE
MORE OF A LISTENING TOUR BECAUSE
I'M STRUCK WITH THE ENGAGEMENT
SO THANK YOU.
THE ONE THING I PARTICIPATED IN
WAS THERE WAS A LOT OF DIVERSITY
IN THE TYPES OF STAKEHOLDERS IN
THE BREAKOUTS I WAS A PART OF.
IT WASN'T THE USUAL SUSPECTS AS
IF YOU WERE TO HAVE AN IN PERSON
NCATS MEETING SO HOI THAT WAS
ANNOUNCED WAS EFFECTIVE AND
THERE WERE DIFFERENT IDEAS AND
DISCUSSIONS COMING FORWARD AND I
WANT TO COMMEND THE FACILITATOR
FOR MANAGING AND WELCOMING ALL
COMMENTS AND GIVING SPACE FOR
EVERYBODY TO EXCHANGE THEIR
IDEAS.
THE ONE THING THAT STRUCK ME IS
THE IDEAS AND SUGGESTIONS COMING
IN OPENED THE DOOR FOR
OPPORTUNITY FOR NCATS TO
ELEVATE, OPTIMIZE, SHINE A LIGHT
ON THE EXISTING RESOURCES PART
OF THE STRATEGY PLANNING OR
STRATEGIC DOING WHICH I THINK
WAS FRAMED IN SOME OF MEETINGS
DOESN'T MEAN WE HAVE TO START
OVER OR BUILD ON NEW.
THE RESOURCES NCATS ALREADY HAS
THAT CAN BE UPDATED OR
REPURPOSED OR GIVEN NEW LIFE
THAT BECAUSE OF THE LISTENING
TOURS YOU'RE REACHING A BROADER
AUDIENCE NOW WHO MAY NOT BE
FAMILIAR WITH SOME OF WHAT IS
ALREADY HERE.
THIS IS THE OPPORTUNITY TO
REPACKAGE IT AND PUSH IT BACK
OUT TO A BROADER AUDIENCE.
TO THINK ABOUT THAT A LITTLE BIT
AND TO PARTNER WITH SOME OF US
IN THINKING HOW TO DO THAT.
THAT WOULD BE ONE COMMENT.
THE OTHER IS I WAS LISTENING TO
YOUR PRESENTATION JUST A LITTLE
BIT AGO AND THINKING ABOUT THE
WONDERFUL TOUR WE HAD THERE'S A
LOT OF WORK YOU'RE DOING TO
CAPTIVATE PEOPLE EARLIER
UPSTREAM IN THEIR CAREERS.
THE PROGRAM K-12 EFFORTS TO
BRING EARLY SCIENTISTS IN AND
MAYBE YOU'RE DOING IT AND
LISTENING TOWARDS ASKING ANY OF
THOSE GROUPS.
ANYONE IN MEDICAL SCHOOLS OR
GRAD SCHOOL PROGRAMS WHO ARE NOT
YET BEING SERVED OR FUNDED
THROUGH NCATS.
SOME OF QUESTIONS.
THESE WILL BE THE STAKEHOLDERS
WE WANT IN THE ENGAGEMENTS AND
MAYBE DOING A ROUNDTABLE WITH
FOLKS WHO AREN'T YET FUNDED BY
NCATS WHO MAY BE PART OF THE
INTRAMURAL/EXTRAMURAL PROGRAM
AND MAY BE A DISTINCT LISTENING
TOUR.
AND IN THE LAST DISCUSSION NOT
JUST THE HEALTH CARE SETTING
WITH PROVIDERS BUT PAYERS AND
THEY'RE THE GATE KEEPERS FOR
WHAT PROVIDERS TALK ABOUT AND
TALKING ABOUT PAY AS AN
INTERESTING GROUP THERE AS WELL.
>>THAT'S A GOOD POINT.
I AGREE PAYERS ARE IMPORTANT.
WE'RE STARTING TO INTERSECT A
LITTLE BIT MORE WITH THOSE
GROUPS ESPECIALLY IN THE WORK
WE'VE BEEN DOING WITH THE AREAS
THAT ARE THE POINTS AND CRITICAL
TO US.
I LIKE THAT DISCUSSION.
I WOULD LOVE TO HEAR HOW TO
INTERACT WITH THE YOUNG FOLKS
OUT THERE.
THOSE WHO ARE MAYBE HIGH SCHOOL
AND COLLEGE AND HOW TO TALK TO
THEM ABOUT WHAT WE DO AT THE NIH
AND THERE'S A CAREER FOR THEM IN
SCIENCE THAT CAN BE FULLY
SUPPORTED WITH EXCITING AREAS OF
ACTIVITY AND WE HAVE PROGRAMS
FOR WOMEN IN SCIENCES AND TAKE
YOUR CHILD TO WORKDAY AT NIH AND
SOMETIMES THE TOUR IS THE
BIGGEST HIT OF THAT.
HOW DO EXPAND THAT IN RURAL
COMMUNITIES AND INTERSECT WITH
PEOPLE WHO AREN'T NECESSARILY IN
THE AREA AND BRING THIS TO THEM
IN A WAY THAT'S MEANINGFUL.
I THINK YOU'RE RIGHT.
BRAIN STORMING ABOUT THAT WOULD
BE GREAT.
AND THEN THE IDEA OF UPDATING
AND REPURPOSING THE RESOURCES I
THINK IS ANOTHER AREA.
WE DON'T WANT TO STOP AND SAY
WE'RE ALREADY DOING THAT OF
COURSE DURING THE ROUNDTABLES
BECAUSE MAYBE PEOPLE DON'T KNOW
SOME OF THEM BUT THE IDEA IS HOW
CAN WE MAKE THAT MORE EASY FOR
THEM TO GO TO A CENTRAL LOCATION
TO FIND OUT MORE ABOUT THE
RESOURCES AND HAVE THEM BE IN A
WAY THEY CAN USE THEM?
I THINK WE HAVE ALL OF THE
THINGS IN PLACE BUT WE NEED TO
TAKE ANOTHER LOOK AT THIS AS
WELL.
I THINK THAT'S A GREAT POINT.
THANK YOU, ANNIE FOR THE
COMMENTS.
GREAT TO SEE YOU.
SERGIO AND THEN MATTIAS.
>>THANK YOU.
I WANT TO THANK YOU, JONI, AND
OTHERS TO USE AN HOUR OF THE
COUNCIL MEETING TO TAKE A DEEP
DIVE.
I APPLAUD THE EFFORT AND AS I
SAID PREVIOUSLY, THE LISTENING
TOURS THAT YOU ARE TALKING ABOUT
I THINK IT'S SO IMPORTANT TO
HAVE A FINGER ON THE PULSE WITH
THE PUBLIC AND DIFFERENT
STAKEHOLDERS.
I APPRECIATE THAT VERY MUCH.
I ALSO PARTICIPATED IN ONE OF
THE ROUNDTABLES AND SPECIFICALLY
IN THE DEIA BREAKOUT AND THE
COMMUNITY ENGAGEMENT I THINK YOU
DID A GOOD JOB SUMMARIZING WHAT
TRANSPIRED AND THE MULTIPLE
COMMENTS.
THERE'S THINGS I WANT TO
EMPHASIZE.
ONE IS RELATED TO THE DIVISION
YOU MENTIONED.
IT'S VERY GOOD BY THE WAY, MORE
TREATMENTS TO ALL PEOPLE MORE
QUICKLY.
AND IN TERMS OF THE OLD PEOPLE,
ONE OF THE THINGS THAT WAS
DISCUSSED AND DIDN'T RISE LIKE
BUILDING TRUST AND BECOMING
TRUSTWORTHY.
BECOMING A TRUSTWORTHY
ORGANIZATION IN WHICH OLD PEOPLE
ESPECIALLY THOSE WOULD HAVE BEEN
UNDER SERVED OR LIVE IN THE
RURAL AREAS THAT USUALLY DON'T
BENEFIT FROM THE GOOD THIS GROUP
AND THE ORGANIZATION DOES.
I WOULD INVITE YOU TO REALLY
THINK THAT THROUGH.
THERE'S A TREMENDOUS BRIDGE WITH
NCATS AND GETTING THE WORK TO
MORE PEOPLE MORE QUICKLY.
THE AND WE CONTINUE TO THINK
THAT TREATMENTS ARE GOING TO
BE -- PEOPLE ARE GOING ACCESS
TREATMENT COMING TO WHERE THE
TREATMENTS ARE.
I CALL THAT THE WAITING MODE.
HERE WE ARE.
YOU CAME TO US.
INSTITUTIONAL BARRIERS AND
STRUCTURAL BARRIERS AND
PERCEPTUAL BARRIERS ESPECIALLY
HISTORICALLY UNDER SERVED
COMMUNITIES HAVE SO I THINK IT'S
TIME WE START THINKING ABOUT
MORE ABOUT THIS AND I THINK IT'S
IMPORTANT TO CONSIDER THE HEALTH
SERVICES FOR EXAMPLE THAT IN
THOSE TREATMENTS THAT ARE BEING
DEVELOPED MORE QUICKLY THAT CAN
BE PROVIDED IN A MORE EFFECTIVE
MANNER OVER TIME WITH AREAS OF
ACCESS TO SERVICES AND I HAVEN'T
SEEN THAT IN THE SUMMARY THAT
YOU MENTIONED.
AND THAT WAS DISCUSSED IN THE
BREAKOUTS THAT WE HAVE.
SO THIS IS JUST A REMINDER THAT
WAS DISCUSSED AND I HOPE IT
MAKES IT.
THE OTHER PIECE IS MENTAL
HEALTH.
MENTAL HEALTH IS USUALLY -- NOT
PUBLIC HEALTH.
I THINK THAT GETS LOST AND MORE
THAN EVER WE OUGHT TO BE
FOCUSSING MORE ATTENTION TO
MENTAL HEALTH.
AND FOCUSSING ON YOUTH AS WELL.
WE CAN COME UP WITH WAYS TO
ENGAGE IN THE PROCESS AS EARLY
AS POSSIBLE WILL SERVE AS WELL
AND IS GOING TO -- I ANTICIPATE
IT WILL WORK WITH THE VISION YOU
HAVE SHARED WITH US.
BRAVO.
IT'S GREAT YOU ARE LEADING THE
EFFORT.
I HAVEN'T SEEN THIS IN OTHER
INSTITUTES FRANKLY OR AGENCIES.
THIS IS PERHAPS UNIQUE.
I DON'T KNOW THE WHOLE LANDSCAPE
BUT THIS IS THE FIRST TIME I
HAVE SEEN THE ROUNDTABLE
LISTENING SESSIONS.
THAT'S GREAT.
>>THANK YOU, SERGIO.
I APPRECIATE THE IDEA OF THE
FACT THAT BUILDING TRUST DOES
NOT EQUAL TRUSTWORTHINESS.
IT'S AN IMPORTANT DISTINCTION.
ONE IS NECESSARY BUT NOT
SUFFICIENT TO HAVE THE OTHER.
AND WITH ALL THE COMMENTS PEOPLE
HAVE MADE GOES TO HOW NCATS CAN
BE MORE TRUSTWORTHY.
I APPRECIATE THE AMALGAMATION
AND WITH COST AND ACCESS THERE
ARE SOME THINGS WE CAN DO AND
SOME THINGS WE SHOULD RESEARCH
MORE.
THERE ARE RESEARCH QUESTIONS WE
COULD BE THINKING ABOUT AND
BUILDING THEM INTO THE FABRIC
AND WHAT THOSE ARE I'M NOT SURE.
THESE ARE AREAS WE NEED TO START
TO EXPLORE MORE IN DEPTH AND
WITH THAT PERHAPS WE CAN START
TO GET AT INTERESTED YOUNG
PEOPLE AND FOLKS WHO ARE GOING
TO BE ABLE TO USE THAT
ENTHUSIASM IN AREAS LIKE MENTAL
HEALTH AND OTHER AREAS.
YOU MAY THINK MENTAL HEALTH IS
NOT IT BUT THERE'S A NEED IN THE
SPACE AND THE NEXT QUESTION IS
HOW WE PRIORITIZE ABOUT THIS.
THINK ABOUT THAT.
BEFORE WE GO THERE I WANT TO GO
TO MATTIAS WHO HAS BEEN WAITING
PATIENTLY.
>>I HAVEN'T SEEN THAT AND YOU
MAY REMEMBER AT THE LAST COUNCIL
MEETING WE DISCUSSED THE
CHALLENGE AND THE ADVANTAGE CTSA
DID NOT REACH THE PATIENT.
WE SHOWED A SAD EXAMPLE THIS
AFTERNOON.
SO YES, PLEASE FOCUS AND ONE
STORY I LEARN FROM AND YES WE
MANAGED NCATS WITH THE
RELATIONSHIPS BETWEEN PATIENT
ORGANIZATIONS AND NCATS AND
THERE IS ONE MODEL FOR APPROACH
TO IT.
AND THE MEETING I STEPPED OUT IS
DOING THAT AND KIDNEY DISEASE
AND INSURANCE COMPANIES ARE
CHANGING THEIR ROLE AS WE SPEAK.
THEY ARE HEADED INTO
IMPLEMENTATION SCIENCE TO REDUCE
COST AND IN THE PAST THE MODEL
BETWEEN CARE PROVISION AND
INSURANCE IS NOT SUFFICIENT OF
THE FUTURE AS BUILDING OUT THE
RESEARCH INFRASTRUCTURE WE CAN
INTERSECT WITH THE HUGE
DATABASES AVAILABLE AND BRING
THEM TOGETHER WITH OUR RESEARCH
EFFORTS I THINK IS A SIGNIFICANT
OPPORTUNITY AND AGAIN CAN
HOPEFULLY BUILD TRUST BACK INTO
A SYSTEM AND THE FINAL POINT
SPEAKING WITH A GERMAN ACCIDENT,
YES, NCAT HAS IMPACT AND
CLAWABLE EFFORTS CAN BE
LEVERAGED FOR INVESTMENT OF TAX
DOLLARS FOR US HERE.
I HAVE NOT SEEN IN THE OUTREACH
EFFORT ANY EFFORTS TO ENGAGE AND
THERE'S AN EXAMPLE HOW NCATS IS
IMPACTING THE RARE DISEASE
GLOBAL COMMUNITY AND HAS TAUGHT
THE COMMUNITY A LOT AND THINKING
OF WAYS HOW TO MAKE SURE THAT
THAT OPPORTUNITY IS NOT LOST.
THANKS SO MUCH FOR ALL YOU DO.
>>THANK YOU SO MUCH.
IS THAT WAS GREAT.
THIS IS MY BAD.
ONE THING I DIDN'T MENTION IN
TERMS OF THE EFFORTS IN THE MORE
INTERNATIONAL SPACING.
"HAVE HAD AN OPPORTUNITY TO
INTERACT WITH SOME OF THE
EMBASSIES IN D.C. AND THE
ATTACHES IN THE SCIENTIFIC
COMMUNITY AND EMBASSIES.
I'LL BE SPEAKING WITH THEM AT
THE SCIENCE DIPLOMAT COUNCIL IN
THE NEXT MONTH AND LOOK FORWARD
TO THOSE CONVERSATIONS AND
BRINGING UP THESE KINDS OF IDEAS
IN THE RARE DISEASE SPACE FOR
EXAMPLE WHERE WE HAVE AN
OPPORTUNITY TO LEVEL THE ABILITY
AND BRING IN MORE OF THE RARE
DISEASE PATIENTS WHO ARE CRASS
THE GLOBE AND MAXIMIZING OUR
ABILITY TO REACH THE PATIENTS IN
THE RARE DISEASE SPACE WHEN IT'S
SO CRITICAL WE DO SO.
WE HAVE PJ BROOKS AS A MEMBER
AND I SHOULD ALSO MENTION YOU'LL
PROBABLY HEAR MORE ABOUT THIS IN
THE UPDATE IN THE DIVISION OF
RARE DISEASE RESEARCH
INNOVATIONS.
WE'LL TALK ABOUT THE RBCRN AND
PJ WILL TELL YOU MORE ABOUT WHAT
WE'RE DOING AND WE'LL TALK ABOUT
SOME OF THOSE IMPACTS BUT
THEY'RE MOBILE IMPACTS.
IT'S CRITICAL AND I'LL TRY TO
HIGHLIGHT THOSE A BIT MORE AND
IT'S A GOOD POINT.
PAUL AND THEN KEITH.
>>I LIKE THE FORMAT.
AND IT FELT LIKE THE PROGRAM WAS
LISTENING.
AND THERE WAS AN ANONYMOUS WAY
TO ANSWER AND AN IDENTIFIED CHAT
WINDOW.
I DON'T THINK PEOPLE WERE
NECESSARILY TRYING TO BE
ANONYMOUS BUT I THOUGHT IT WAS
NICE THEY WOULD HAVE THE OPTION
IF THEY WANTED.
GOING BACK TO ANDY KENNEDY'S
COMMENTS, I AGREE.
THE LISTENING SESSIONS ARE NOT
THE TIME TO SAY YOU DIDN'T KNOW
THIS EXISTED BUT IT WOULD BE
GREAT AS YOU WRAP UP THE
SESSIONS MAYBE PUTTING A
COMMUNICATION OUT ABOUT WHAT YOU
HEARD AND HOW TO USE THE
INFORMATION WOULD BE A GREAT
TIME TO HIGHLIGHT THINGS ALREADY
THERE.
OF THOSE THINK CONTINUOUS SMALL
COMMUNICATIONS WOULD BE A GREAT
WAY TO KEEP FRONT AND CENTER AND
IN PEOPLE'S MINDS AS DIFFERENT
ASPECTS OF THE NCATS PROGRAM AT
LARGE.
>>I AGREE KEEPING THE
COMMUNICATION PIECE OPEN AND
GOING FORWARD AND TALKING ABOUT
THE SUCCESSES AND PERHAPS EVEN
WHAT'S NOT WORKING WELL AND THAT
DISSEMINATION PIECE OF EVEN THAT
I THINK WILL BE INTERESTING.
WE'VE TALKED ABOUT THIS A BIT
AND WITH THE AMPLIFICATION OF
WHAT YOU SAID AND INTERNALLY IT
TELLS US IT'S PROBABLY A
DIRECTION WE CAN TRY TO MOVE IN.
UP IN
>>AS I THINK ABOUT COMMUNITY
ENGAGEMENT AND ENGAGEMENT OF
PATIENTS.
A PARALLEL STRIKES ME FROM THE
TOUR TODAY WHEN WE SAW THE
DEMONSTRATION OF TRYING TO MOVE
FROM THE EXPERIMENTS THAT DON'T
ENGAGE PATIENTS DIRECTLY BUT
MAYBE IN THE CLINICAL TRIAL
SPACE AND TO THE FIELD AND THE
INFUSION OF THE CELL INTO MUSCLE
OR SKIN TISSUE TO SEE WHAT'S THE
REAL WORLD IMPACT.
I THINK ABOUT THE SAME THING
WHEN IT COMES TO DISSEMINATION
AND TRYING TO ACHIEVE AGAIN THE
VISION TREATMENT FOR ALL PEOPLE
MORE QUICKLY.
HOW DO YOU GET TO THE COMMUNITY
LEVEL NOT EVEN JUST THE
CLINICIANS BUT INTO THE ENTIRE
COMMUNITY.
I THINK OF TWO SPECIFIC EXAMPLES
TO DO THAT.
ONE IS FOR DIRECTED ENGAGEMENT
OF THE SOCIAL SCIENTISTS WHO
UNDERSTAND THAT DYNAMIC MORE AND
THE CTSA PROVIDE AN EXCELLENT
PLATFORM FOR DOING IT AND THE
OTHER IS PLAYING OFF THE
TELEHEALTH DISSEMINATION IDEA
BECAUSE WE WORK ON THAT AND THE
PANEL I SHARED AND I JOTTED DOWN
A COUPLE EXAMPLES WHY IT'S
IMPORTANT TO UNDERSTAND THE END
POINT AS BEST YOU CAN.
ONE IS IS IT EVEN AVAILABLE.
WE'RE STILL TRYING TO EXPAND THE
ACCESS TO BROADBAND TECHNOLOGY
IN A WAY IF WE START THINKING
ABOUT DIGITAL TECHNOLOGY IN THE
HOME THERE'S A LARGE PERCENTAGE
OF AMERICA WHERE THAT'S NOT
POSSIBLE YET AND THE SECOND ONE
I'VE ONLY MORE RECENTLY BEGAN TO
THINK ABOUT MORE CAREFULLY IS
DIGITAL LITERACY.
IF YOU TRY TO APPROACH A
POPULATION THAT IS BEHIND THE
TIMES AND THE AGE COHORT MANY
COLLEAGUES AND I ARE IN AND SAW
A GOOD CASE STUDY OF THIS AT THE
NATIONAL HEALTH ASSOCIATION LAST
WEEK A PROJECT IN MINNESOTA
CALLED REACH WHERE THEY'RE
REACHING INTO THE LOCAL
COMMUNITIES THROUGH THE LIBRARY
TO HELP THE ELDERLY POPULATIONS
WITH DIGITAL LITERACY AS WELL AS
ACCESS TO BROADBAND.
THAT'S THE INNOVATION ON THE
SOCIAL SIDE THAT WOULD HELP
NCATS AND OTHERS MOVE THE
CLINICAL DEVELOPMENT TO THE
PATIENT AND A BROADER CROSS
SECTION OF PATIENTS.
>>KEITH, AND GOING BACK TO
SERGIO WHEN I SHOWED THE PIECE
ON THE COMMUNITY ADVISORY BOARD
MEMBER IN THIS PARTICULAR
PROJECT WITH THE NATIONAL
ACADEMIES THE IDEA OF THE SOCIAL
PIECE THE SOCIAL COMMUNITY
ENGAGEMENT WHAT SHE FOUND WAS
GOING INTO INTO THAT SCENARIO
SHE HAD A PRECONCEPTION BUT
AFTER ENGAGEMENT FIRST IT'S NOT
THE ROOT SHE TALKS ABOUT HER
ABILITY TO PIVOT TO WHAT THE
ROOT OF THE PROBLEMS AND WHEN
YOU TALK ABOUT ENGAGING SOCIAL
SCIENTISTS TO THINK OF THAT
ENGAGEMENT AT THE COMMUNITY
LEVEL OFTEN WE THINK ABOUT THAT
AS BEING UNTANGIBLE OR HARD TO
DO, DIFFICULT.
IT'S NOT SOMETHING WE KNOW A LOT
ABOUT HOW TO DO BUT THE SOCIAL
SCIENTIST ENGAGING THEM AND THE
IMPORTANCE OF DISSEMINATING
CRITICAL WORK WE'RE DOING AND
MAKING PEOPLE AWARE HOW TO START
THROUGH THE HEALTH LITERACY
PROGRAMS AND I WAS STRUCK ABOUT
THE VIDEO I SHOWED IS THE
SOLUTIONS AREN'T OFTEN THAT
CHALLENGING BUT WE DON'T TAKE
THE TIME TO UNDERSTAND THEM.
AND IN THE ENVIRONMENTS IS WHERE
THE MAGIC CAN HAPPEN.
WHAT YOU'RE SPEAKING TO IS THE
IDEA OF HOW TO ENGAGE THAT PIECE
AND GET INTO THE SOCIAL
CONSTRUCT OF WHAT WE'RE TALKING
ABOUT EVEN BETTER.
CERTAINLY TELEHEALTH AND
TELEMEDICINE IS USED AT A LEVEL
OF HEALTH CARE AND IN TERMS OF
HEALTH LITERACY AND DIGITAL
LITERACY THERE ARE LOTS OF
OPPORTUNITIES THERE WE HAVE A
PROGRAM AND CHRIS RUNS OUR
DIGITAL HEALTH ASPECTS AND SO I
THINK THIS WILL BE EXPECT THAT
WILL CONTINUE TO TALK ABOUT MORE
AND SO I APPRECIATE YOUR
COMMENTS ON THAT AND FIGURING
OUT HOW TO WORK WITH THE SOCIAL
SCIENTISTS TO GET MORE INSIGHT
INTO THAT.
THAT'S A GREAT IDEA.
THANK YOU.
I DON'T SEE ANY OTHER HANDS.
AM I MISSING ONE?
GO AHEAD.
>>I WANT TO SPRING BOARD ON THE
LAST DISCUSSION WITH THE
EXTENSION AGENTS AND USUALLY RUN
THROUGH THE LENS OF A UNIVERSITY
AND MY MOTHER WAS AN EXTENSION
AGENT AND THEY KNOW THE
COMMUNITY AND HAVE THE TRUST OF
THE PEOPLE IN THE COMMUNITY AND
WE'RE NOT EQUALLINGIZING THEM
FOR THIS KIND OF STUFF AND THEY
HAVE A FOOTPRINT IN EVERY
COUNTY.
OFTEN A LOT OF THEM IN THE 4H
PROGRAM AND RURAL COMMUNITIES.
AROUND MEDICINE HEALTH AND
SCIENTISTS IN YOUR LOCAL
COMMUNITY AND THE LEVELS THAT
CAN BE DONE TO ENGAGE BEFORE THE
10 YEARS OF AGE.
THESE ARE PROGRAMS THAT HAVE AN
INFRASTRUCTURE THAT WE JUST NEED
TO TAP INTO.
>>GREAT POINT.
AND MIKE IF YOU'RE STILL AT THE
TABLE MAYBE YOU CAN COMMENT ON
THIS AS WELL.
I'LL SAY THROUGH THIS WAS
PROBABLY PRE-PANDEMIC, I THINK,
BUT WE HAD THE IDEA OF THE
EXTENSION PROGRAM AND THINKING
OF WAYS TO ENGAGE AND THE MAY BE
TIME TO PICK UP ON THAT AGAIN.
I'M ALSO FROM A RURAL COMMUNITY
IN KANSAS AND GREW UP WITHIN
EXTENSION PROGRAMS.
WHEN I THINK ABOUT IT, I THINK
ABOUT IT IN THE AGRICULTURE
SECTION BUT THERE'S A LOT OF
WORK IN THE HEALTH SPACE TOO AND
HOW TO TAP IN MORE WOULD BE
GREAT.
THE OTHER POINT IS WE'VE BEEN
STARTING TO ENGAGE WITH HHRQ AND
THE HEALTH RESEARCH AND QUALITY
AND THINKING ABOUT AREAS WHERE
THEY'RE SUPPORTED IN THIS
SIMILAR VEIN AS WELL.
I WANTED TO HAVE YOU COMMENT ON
THE CTSA MEETING.
>>SO PRE-COVID WHICH I
RECOGNIZE WAS A LONG TIME AGO WE
HAD A MEETING ON RURAL HEALTH
AND THE TOPIC OF THE EXTENSION
SERVICE CAME UP BECAUSE MANY
ACTIVELY ENGAGE WITH THEM AND IT
WAS SURPRISING BECAUSE MANY WERE
TOTALLY UNAWARE OF THIS.
I HAD A COUPLE FOLLOW-UP
MEETINGS WITH OFFICIALS FROM THE
USDA AND INVITED ONE TO COME TO
OUR ANNUAL PROGRAM MEETING TO
MAKE THIS AVAILABLE.
TALK ABOUT IT.
I DON'T KNOW THE BARRIERS THAT
EXIST IN INDIVIDUAL STAKES THAT
MAY PRECLUDE THOSE INTERACTIONS
FROM OCCURRING.
WHEN THEY DO OCCUR WE HAVE MANY
EXAMPLES ACROSS THE CTSAs WHERE
THEY'VE BEEN PRODUCTIVE AND FROM
THE USDA PERSPECTIVE IN TALKING
ABOUT THE AGRICULTURAL EXTENSION
SERVICE IS HISTORICALLY
SOMETHING WHERE FARMERS WOULD
COME IN AND SAY CAN YOU ANALYZE
MY SOIL AND TALK ABOUT
FERTILIZER OR PESTICIDES AND ALL
THAT STUFF.
WHAT THE USDA OFFICIALS ARE
SAYING IS THE AGRO BUSINESSES DO
THAT AND SO THEIR FOCUS IS ON
THE HEALTH OF THEIR COMMUNITIES
BECAUSE THAT'S WHAT'S SUFFERING
AND ONE INDIVIDUAL SAID PEOPLE
TALK ABOUT FOOD DESERTS IN
CITIES IN RURAL COMMUNITIES WE
HAVE FOOD SLUWAMPS AND A DIFFERT
ISSUE.
THEY ARE FOCUSSED AND HAVE THE
CONNECTIONS AND THE ENGAGEMENT
AND TRUST OF THE COMMUNITY.
SO IT'S SOMETHING WE WERE
STARTING TO WORK ON PRE-COVID
AND COVID PUT A LOCK ON A LOT OF
ACTIVITIES BUT IT'S SOMETHING WE
NEED TO GET BACK TO BECAUSE IT'S
A VALUABLE ADDITION TO THE CTSA
TO HELP THEM AMPLIFY WHAT TO DO
IN THEIR REGION.
>>THANK YOU FOR THAT COMMENT,
PAULA.
I'M RECOGNIZING I THINK WE'RE A
LITTLE BIT OVER TIME, ANNA, HOW
ARE WE DOING?
ARE WE READY FOR A BREAK?
>>YES, ACTUALLY.
WE'RE A FEW MINUTES PAST SO
WE'VE GOT TIME ON THE BACK END
SO YES, TIME FOR A BREAK.
>>PERHAPS WE CAN PICK UP SOME
MORE OF THIS DISCUSSION MORE AT
THE END OF THE DAY IF THERE'S
TIME.
AND IF NOT, JUST FAIR WARNING
YOU'RE PROBABLY NOT GOING TO BE
OFF THE HOOK JUST YET.
I'LL PROBABLY REACH OUT MORE AND
THANK YOU IN ADVANCE BUT I ALSO
THANK YOU NOW FOR ALL THE INPUT
YOU HAVE GIVEN US FOR THESE
ADDITIONAL SUGGESTIONS AS WE
START TO FINAL I'D AND ROUND
THIS OUT WE HAVE A LITTLE BIT
MORE WORK TO DO BUT I THINK IT'S
REALLY EXCITING AND WITH THAT I
THINK WE CAN BREAK NOW AND ANNA
WOULD SHOULD THEY BE BACK?
>>LET'S SAY 3:25.
>>SOUNDS GOOD.
SEE YOU SOON.
THE FIRST PRESENTATION IS FROM
THE DIVISION OF CLINIC
INNOVATION AND WILL
INTRODUCE THE TALK FROM THEIR
PERSPECTIVE AND I'LL LET YOU
INTRODUCE DR. MATHE.
>>DIRECTOR FOR INFORMATICS AT
THE NCATS SHE JOINED FROM OHIO
STATE UNIVERSITY SHE STARTED
AFTER COVID INSTRUCTIONS WERE
IMPROVED.
DESPITE THIS CHALLENGE SHE WAS
ABLE TO ORGANIZE HER TEAM TO
PRODUCE TRANSFORMATIVE WORK NOT
JUST IN RESPONSE TO THE PANDEMIC
THE SARS COV2 INFECTION WORK AND
THE FLOOR IS YOURS.
>>I'LL SHOW YOU SOME OF THE
WORK WE'VE BEEN DOING IN THE
INFORMATICS AND WE REFER TO IT
AS ISS.
FIRST I'M GIVE YOU BACKGROUND
INFORMATICS AND THE DIVISION OF
PRECLINICAL INNOVATION
INFORMATICS CORPS AND HOW THIS
RELATE TO NCATS.
OUR MISSION AND GOALS AND
CULTURE AND THEN GO INTO THREE
DIFFERENT AREAS WE'VE DONE.
THE FIRST RELATED TO COVID-19
AND AS AN EXAMPLE HOW WE'VE BEEN
ABLE TO PUSH THE BOUNDARIES OF
INFORMATICS AND I'LL ALSO TALK
ABOUT WAYS IN WHICH WE DERIVE
ACTIONABLE INSIGHTS BY
INTEGRATING DIFFERENT TYPES OF
COMPLEX DATA.
AND FINALLY I'LL TOUCH ON NEW
TECHNOLOGIES THAT WE'RE BRINGING
TO NCATS AROUND METABOLOMICS AND
MULTIOMICS.
WHEN WE THINK OF DATA I THINK WE
ALL KNOW THERE'S A LOT OF DATA
COMING AT US.
THE TOP LEFT STICK TURE IS AN
OLD CART FROM THE ECONOMIST IN
2010.
IT SHOWS THE DATA DELUGE BEING
FUNDED BY AN UMBRELLA AND
INTERPRETED FOR THE FLOWER WHICH
CAN BE THOUGHT OF THE ACTIONABLE
INPUT.
I ALWAYS REFER TO THIS AND THINK
ABOUT IT BECAUSE THERE'S STILL A
LOT OF DATA COMING AT US AND IF
YOU LOOK AT THE BOTTOM LEFT
BEFORE THE '60 WE HAD
QUALITATIVE AND THEN
QUANTITATIVE AND WE SAW A
TECHNOLOGY REVOLUTION WHERE WE
WERE ABLE TO MEASURE MORE THINGS
IN SPECIFIC WAYS.
BETWEEN THE '80s AND 2010 WE SAW
AN EXPANSION OF THESE
TECHNOLOGIES BEING APPLIED IN
DIFFERENT APPLICATIONS IN
DIFFERENT AREAS AND BEYOND 2010
THERE'S A LOT OF AUTOMATION AND
WE ALL KNOW NCATS PLAYS A KEY
ROLE HERE IN BEING ABLE TO
AUTOMATE AND GENERATE HIGH
QUALITY ROBUST DATA AT A LARGE
ZAL.
-- SCALE.
THIS IS GREAT.
NOW WE HAVE A LOT OF DATA BUT
THE DATA TYPES AND THE
COMPLEXITY OF THE DATA ARE
INCREA
INCREASING CONDUCTING
LARGE-SCALE STUDIES ARE DATA
EXTENSIVE AND HARD TO EXTRACT
MEANING FROM ALL THE DATA AND
DERIVE THE ACTIONABLE INSIGHT.
TODAY I'M GOING TO TALK ABOUT
HOW AND DISSEMINATING DATA AND
ANALYZING DATA USING THE TOOLS
WE GENERATE IN HOUSE BUT ALSO
TOOLS THAT WE GENERATE IN
COLLABORATION WITH OTHER
EXPERTS.
AS A REMINDER THE MISSION IS TO
TRANSFORM DISCOVERY APPROACH AND
TOOLS, ADVANCE TEAM SCIENCE AND
CATALYZE THE BIO MEDICAL
COMMUNITY TO DELIVER MOST
EFFECTIVE THERAPIES TO TREAT
HUMAN DISEASES.
WITHIN THIS OUR MISSION IS TO
DERIVE ACTIONABLE INSIGHT FROM
INTEGRATING TRANSLATIONAL
RESEARCH DATA AND TO ACCELERATE
TRANSLATION OF FINDINGS INTO THE
CLINIC.
SO CONCEPTUALLY WHAT THIS MEANS
IF YOU LOOK AT THE BOTTOM HERE
WE LOOK AT VARIOUS TYPES OF DATA
FROM BASIC PRE CLINICAL TO
CLINICAL DATA AND THE DATA COME
IN MANY DIFFERENT TYPES OF
FLAVORS.
SOME COULD BE EXPERIMENTAL
MEASUREMENTS AND IT COULD COME
FROM LITERATURE, SOCIAL MEDIA
AND ETCETERA.
AND WE INTEGRATE ALL THE
INFORMATION AND WE GET THIS AND
THEN WE ANSWER SPECIFIC
QUESTIONS AND YOU CAN SEE AN
ORDER OF PATTERNS.
AND ACTIONABLE INSIGHT WHICH AT
THE END OF THE DAY WE WANT TO
REACH.
A LITTLE BIT ABOUT THE RESEARCH
AREAS.
INFORMATICS WAS ONE OF THE
BRANCHES OF THE EARLY
TRANSLATION BRARCHLG AND --
BRANCH AND IT WAS INFORMATICS
HEAVY.
THEN IN 2021 TO NOW IT'S
EXPANDED TO DIFFERENT AREAS AND
SOME INCLUDE MOLECULAR PROFILING
AND MOLECULAR EPIDEMIOLOGY WORK.
THE NEW AREAS WE DELVE INTO COME
TO THE HISTORICAL SCALE AND THE
ONGOING EFFORTS IN THE FUTURE
EFFORTS THAT WE'RE PLANNING.
IF YOU LOOK AT THE LEFT YOU CAN
SEE AREAS IN TEAL IDENTIFYING
MECHANISMS AND TARGETS IN
MODELS, TESTING CHEMICALS AND
ASSAYS AND PUBLICLY
DISSEMINATING THE RESULTS, ARE
ALWAYS PART OF IFX.
THEY STILL ARE TODAY.
THE NEW AREAS THAT WE'RE DELVING
IN IS DEVELOPING NEW ASSAY TYPES
I'LL TALK BAND ASSESSING
MECHANISMS AN TARGETS IN
POPULATION IN ADDITION TO MODELS
AND IDENTIFYING PREDICTIONS.
OF COURSE ALL THESE EFFORTS FALL
UNDER THE UMBRELLA OF USING
INFORMATION SO THEY CAN INFORM
CLINICAL TRIALS AND FDA
APPROVED.
THERE'S TEAM SCIENCE IN OUR
WORK.
WE HAVE EXPERTS IN INFORMATICS
AND DIFFERENT OMICS, MACHINE
LEARNING, SOFTWARE DEVELOPMENT
AND ALSO PROJECT MANAGEMENT
WHICH I PERSONALLY VALUE VERY
MUCH.
IT WAS KNEW TO ME WHEN I CAME
HERE AND HAD THE LUXURY IN
ACADEMIA.
OF COURSE WITH COLLABORATE WITH
OTHER NCATS AND THERE'S IN
BIOLOGY, CHEMISTRY AND THERE'S
MANY EXPERTS BUT THE POINT IS
THAT EVERYBODY TALKED TO EACH
OTHER PRETTY EFFICIENTLY BECAUSE
WE KNOW ENOUGH ABOUT THE OTHER
FIELD TO FIND WAYS OF
COMMUNICATING AND I THINK THAT'S
IMPORTANT.
THE ABILITY TO BE MULTILINGUAL.
WE MAY NOT BE EXPERTS IN SORN
AREAS BUT KNOW ENOUGH TO BE ABLE
TO CONVERT WITH PEOPLE IN THOSE
FIELDS.
AT THE END OUR GOAL IS TO DEEPLY
UNDERSTAND THE DETE BECAUSE WHEN
WE UNDERSTAND THE DATA HOW IT'S
BEING GENERATE AND WHY IT'S
BEING GENERATED AND THEN WE CAN
INFER THE ACTIONABLE INSIGHTS.
SO NOW I'M GOING DELVE INTO
THREE DIFFERENT AREAS OF WORK
WE'VE BEEN DOING AND STARTING
WITH COVID-19.
AND I'M SURE MANY ARE AWARE OF
THE COHORT COLLABORATIVE AND
THIS STARTED EARLY ON IN THE
PANDEMIC AND I'M SURE ALL OF YOU
REMEMBER THERE WERE REAL TIME
TRACKERS THAT WERE COMING OUT.
THIS I THINK OF AS A REAL-TIME
TRACKING FOR EHR DATA IN
COVID-19 AND IT'S ONE OF THEEST
COLLECTIONS MUCH HARMONIZED
CLINICAL HEALTH DATA IN THE
UNITED STATES.
IT'S HUGE.
IT HAS 76 SITES OVER 18 MILLION
PERSONS AND OVER 7 MILLION COVID
POSITIVE CASES.
ALL THIS DATA IS HOSTED IN A
SECURE DATA ENCLAVE AT NCATS.
I CAN'T STRESS HOW IMPORTANT
THIS IS BECAUSE TO PUT TOGETHER
DATA EVEN FROM TWO DIFFERENT
INSTITUTIONS ON YOUR OWN WITHOUT
THIS HUGE UMBRELLA THAT CAN TAKE
YEARS SOMETIMES TO GET THE
APPROPRIATE AGREEMENTS TO GET
THE APPROPRIATE DATA AND
UNDERSTAND HOW THE DATA IS
GENERATED TO HARMONIZE.
WE'RE USING THIS RESOURCE.
I'M GOING GIVE YOU AN EXAMPLE OF
A STUDY WHICH WAS RECENTLY
ACCEPTED AND WILL BE PUBLISHED
VERY SOON.
THIS STUDY IS LOOKING AT
PRE-EXISTING AUTOIMMUNITY AS A
RISK FACTOR FOR COVID-19.
WE WERE INTERESTED IN THIS
BECAUSE WE WERE LOOKING AT THE
CDC LIST OF RISK FACTORS.
TYPE 1 DIABETES WAS LISTED BUT
NO OTHER AUTOIMMUNE DISEASES
WERE LISTED.
THERE'S A PRECEDENCE FOR
THINKING AUTOIMMUNITY OR
TREATMENT BY IMMUNOSUPPRESSIVE
DRUGS COULD HAVE A STRONG IMPACT
ON THE RESPONSE TO COVID-19.
AND SO OUR GOAL WAS TO DIRECTLY
EVALUATE IN A LARGE POPULATION
RIGHT AT NCC THE IMPACT OF
AUTOIMMUNITY AND THE EXPOSURE TO
IMMUNOEXPRESSIVE DRUGS ON
COVID-19.
IT'S IMPORTANT BECAUSE THE IDEA
IS TO HELP INFORM HEALTH CARE
GUIDELINES AND RAISE AWARENESS
FOR PATIENTS AT RISK.
SO WE WERE ABLE TO GENERATE A
COHORT OF OVER 2.4 MILLION
PATIENTS.
SOME ABOUT 190,000 HAVE
PRE-EXISTED AUTOIMMUNE DISEASE
AND ABOUT 50,000 HAD BOTH AND WE
FOUND THAT ADULTS WITH
PRE-EXISTING AUTOIMMUNE DISEASE
IMMUNO SUPPRESSIVE DRUG OR BOTH
ARE MORE LIKELY TO HAVE MORE
SEVERE DISEASE OUTCOMES.
THE RESULTS ARE CONSISTENT EVEN
WHEN WE ADJUST FOR VACCINATION
STATUS OR ANTI-VIRAL GENES.
ANOTHER LARGE SCALE STUDY
POSSIBLE DURING COVID-19 WHICH I
FEEL WOULD HAVE BEEN A LITTLE
BIT HARDER IN PREVIOUS PANDEMIC
TIMES IS LOOKING AT A LARGE
COHORT OF PATIENTS AND LOOKING
AT THEIR METABOLISM AND SEE
WHETHER THERE'S ANY
CHARACTERISTICS OF THE
METABOLISM THAT PRE-EXPOSES
PATIENTS TO SEVERE COVID.
AND THIS SAY COLLABORATION WITH
HARVARD MEDICAL SCHOOL AND WE
COLLECTED IN-PATIENT PRIOR TO
THEM HAVING COVID AND WE WERE
ABLE TO ACCESS THE SAMPLES
THROUGH A BIO BANK AND ANALYZE
THEM FOR WHAT WE CALL THE
METABOLOME.
THIS IS LOOKING AT ALL THE SMALL
MOLECULES RELATED TO METABOLISM.
AND WE ALSO HAD PLASMA ON
PATIENTS DURING ACTIVE DISEASE
AND AFTER.
WE WERE ABLE TO TRACK WHETHER
THE PREDISPOSITION FACTORS GOT
WORSE DURING DISEASE AND WHAT
HAPPENED AFTER.
THIS STUDY IS PARTICULARLY
IMPORTANT BECAUSE OTHER STUDIES
WERE TRYING TO GET AT THIS BUT
WERE EITHER LIMITED IN SIZE,
LOOKED AT A SINGLE TIME POINT
AND NONE OF THEM HAD A TIME
POINT OF COLLECTION THAT WAS
PRIOR TO COVID.
HERE WE'RE LOOKING AT THE
PREDISPOSITION.
WHAT WE FOUND IS AMINO ACID
NUCLEOTIDES AND SINGLE LIPIDS
ARE ASSOCIATED WITH SEVERE
COVID-19 OUTCOMES.
AND THIS STUDY IS LAYING
FOUNDATIONAL WORK FOR
IDENTIFYING BIOMARKERS AND
LOOKING FOR MOLECULAR FUNCTIONS
THAT PREDISPOSE.
THE THIRD EXAMPLE I WANT TO GIVE
IS GOING BACK TO REAL-WORLD
DATA.
THIS IS AN EFFORT CALLED CURE
I.D.
AND IT HAS INFORMATION ON NOVEL
USES OF EXISTING DRUGS FROM
CLINICIANS AND COMING VERY ZOON
FROM PATIENTS AND THE IDEA IS TO
USE THE DATA TO IDENTIFY DRUG
REPURPOSING CANDIDATES TO
PROVIDE EVIDENCE FOR EXPANDING
DRUG LABELS AND FOR INFORMING
THE DESIGN OF CLINICAL TRIALS.
AND YOU CAN SEE ON THE BOTTOM
LEFT A PICTURE OF THE WEB
APPLICATION WHICH IS ALSO A
MOBILE APP WHERE PHYSICIANS AND
PATIENTS CAN ENTER INFORMATION
SO THIS SAY LARGE COLLABORATION
WE HAD WITH THE FDA AND THE
CRITICAL PATH INSTITUTE AND THIS
WAS SUPPORTED NEW WORK WITH THE
HHS WHICH ENABLED US TO LOOK AT
DIFFERENT WAYS OF INCORPORATING
DATA INTO CURE I.D. AND THAT WAY
IS THROUGH E.H.R.s.
SO THIS IS SIMILAR IN THE SENSE
IT'S E.H.R. DATA BUT THERE'S KEY
DISTINCTIONS.
ONE KEY DISTINCTION IS IT THAT
NATIONAL SITES CAN CONTRIBUTE
DATA AND ANOTHER DISTINCTION IS
THAT THE UNDERLYING DATA MODELS
USED DON'T HAVE TO BE ONE OF
THOSE LARGE COMMON ONES.
SO THOSE SMALL INSTITUTIONS THAT
DON'T HAVE THE LARGE
INFRASTRUCTURE TO HAVE THE
ELECTRONIC HEALTH RECORD WELL
ORGANIZED AND INTEGRATABLE WITH
OTHER INSTITUTIONS, THEY'RE ABLE
TO PARTICIPATE AND THEY ARE.
THE MAJORITY OF THE INSTITUTION
THE SMALLER -- SOME LESS
RESOURCED INSTITUTIONS.
ALL THE DATA HERE IS PUBLIC.
AND SO THERE'S THE RELEASE OF
THIS THAT'S GOING TO BE COMING
OUT SOON.
SOMETHING TO LOOK OUT FOR AND
WHILE THIS WAS BILL -- BUILT
FOR COVID-19 THE INFRASTRUCTURE
SIN PLACE TO BE ABLE TO OPEN --
IS IN PLACE TO OPEN IT UP TO
OTHER DISEASES AND OF COURSE
THIS IS PLANNED.
I'LL SWITCH GEARS A LITTLE BIT
FROM COVID-19 AND HOW WE DERIVED
ACTIONABLE INSIGHT FROM THE
DATA.
MOST ARE FAMILIAR WITH DATA
REPOSITORY AND THEY PROVIDE
ACCESS TO DATA AND FOR EXAMPLE
HOW IS IT COLLECTED AND MAYBE
SOME ASPECTS OF STUDY DESIGN.
THEY HAVE KEY LIMITATIONS THAT
I'D LIKE TO HIGHLIGHT.
FIRST IF YOU'RE TRYING TO DO A
STUDY WHERE YOU'RE TRYING TO
REDUCE EXISTING DATA IT TAKES
QUITE A BIT OF TIME AND
RESOURCES.
THOUGH YOU MAY GET ACCESS TO THE
DATA THE FLAVOR OF THE DATA AND
THE FORMATTING OF THE DATA
DOESN'T EASILY LEND ITSELF TO
PUT DATA TOGETHER.
ALSO ANOTHER KEY LIMITATION IS
DRAWING INFORMATION FROM
MULTIPLE SOURCES SOMETIMES IS
HARD.
DEPENDING ON THE DATA TYPE AND
MAYBE DIFFERENT AGREEMENTS THAT
NEED TO BE PUT INTO PLACE.
OR EVEN IF THE DATA IS PUBLIC
SOMETIMES YOU STILL NEED TO HAVE
ACCESS CONTROLS.
YOU STILL NEED TO GO THROUGH
SOME PAPERWORK AND SUBMISSIONS.
SO WHAT IS NEEDED REALLY IS TO
BUILD OFF WHAT IS KNOWN.
YOU HAVE ACCESS TO
INTERPRETATIONS WE CAN BETTER
UNDERSTAND WHAT THEY MEAN BASED
ON PRIOR KNOWLEDGE.
SO THE IDEA IS TO UPON EXISTING
KNOWLEDGE AND OVERLAY NEW DATA.
HOW DO WE DO THIS?
WE DO THIS BY CONNECTING
DIFFERENT TYPES OF DATA AND
DEVELOPING ALGORITHMS AND
MACHINE LEARNING TOOLS AND
METHODS.
I'LL GIVE EXAMPLES.
WE'LL START WITH STANDARDS AND
INTEROPERABILITY.
AND THE EXAMPLE I WANT TO GIVE
HERE STANDARDIZATION OF
MEDICINAL PRODUCTS.
SO THE GLOBAL SUBSTANCE
REGISTRATION SYSTEM, GSRS WAS
IMPLEMENTED AND THIS IS A
COLLABORATION WITH THE FDA.
THIS PROVIDES PUBLIC ACCESS TO
DATA ON SUBSTANCES FOUND IN
MEDICINAL PRODUCTS.
THOSE INCLUDE SMALL MOLECULES
AND ALSO INCLUDE ANTIBODIES,
THERAPIES, ETCETERA.
AND THIS IS ACTUALLY THE FIRST
SYSTEM THAT IS 0133 STANDARD
COMPLIANCE AND THIS IS DATA TO
BE ABLE TO TRACK AND MAKE SURE
IF YOU'RE CALLING THIS MOLECULE
X, IT'S THE SAME MOLECULE X
SOMEBODY ELSE IS LOOKING AT THAT
MOLECULE.
IT'S REALLY MAKING THE
CONNECTION AND ALL THE IS
CURATED BY THE FDA.
THIS OFFER HAS TAKEN OFF AND
THERE'S NOW BROAD ADOPTION
BEYOND THE FDA.
WE ALSO USE IT INTERNALLY AT
NCATS.
BUT IT'S ALSO ADOPTED BY THE
W.H.O. AND THE EUROPEAN AGENCY
AND UNITED STATES PHARMACOPEIA
AND LEVERAGE THE INFRASTRUCTURE
IN DIFFERENT REGULATORY AGENCIES
AND WITHIN INDUSTRY.
YOU CAN SEE A SNAPSHOT OF WHAT
IT LOOKS LIKE ON THE RIGHT WE
HAVE A NICE REGISTRATION SYSTEM
AND YOU CAN BROWSE THE MOLECULES
BUT SOMETIMES THERE'S
INFORMATION THAT MAY BE MISSING.
FOR EXAMPLE, KEY DATA OR OTHER
BIOLOGICAL ACTIVITY OR OTHER
TYPES OF CLINICAL USES AND SO
HERE WE'VE DEVELOPED INSIGHT TO
DRUGS AND THIS CONTAINS CURATED
INFORMATION FROM THE FDA THROUGH
GSRX AND CONTAINS PUBLIC
INFORMATION DRAWN FROM REPUTABLE
RESOURCES AND ALSO DATA FROM
PRIVATE COMPANIES.
THIS DATA IS CURATED AND SERVED
IN THIS APPLICATION THAT YOU SEE
HERE.
AND THEN OF COURSE THIS DATA CAN
BE BROWSED AND USED IN OTHER
RESOURCES AND WE'RE WORKING ON
ENHANCING THE INTEROPERABILITY
SO PEOPLE CAN GET ACCESS TO ALL
THE DATA.
RIGHT NOW THEY HAVE ACCESS TO
ONLY SOME.
SO I GIVE YOU TWO EXAMPLES.
WE ACTUALLY HAVE QUITE A BIT
MORE AND SOME OF THEM ARE LISTED
HERE.
SO WE HAVE DATA AND APPLICATIONS
FOR INFORMATION RELATED TO DRUGS
AND DRUG SUBSTANCES.
I MENTIONED METABOLISM AND WE
WORK CLOSELY WITH THE DIVISION
OF CLINICAL INNOVATION AND THE
EXTRAMURAL DIVISION ON GUARD AND
HAVE A RARE DISEASE ALERT SYSTEM
WHICH IS NEW.
WE HAVE INFORMATION ON ASSAYS.
SO WE WORK WITH NLM AND THE OPEN
DATA PORTAL AND FINALLY ALL OF
THE EXCEPT THE OPEN DATA PORTAL
HAVE COME AND GUARD ARE LED BY
IFX AND NOW AGAIN GOING BACK TO
THE BIGGER VISION WE HAVE A LOT
OF DATA AT OUR DISPOSAL.
WE UNDERSTAND THE DATA VERY WELL
BECAUSE WE'RE THE ONES PUTTING
IT TOGETHER.
WE'RE INVOLVED IN PUTTING IT
TOGETHER.
SO HOW DO WE NAVIGATE THROUGH
IT?
SO LET ME SHOW YOU AN EXAMPLE IN
THE RARE DISEASE SPACE.
HERE WE ARE AIMING TO CLUSTER
RARE DISEASES AND WE WANT TO DO
THIS BECAUSE WE WANT TO FIND
SHARED CHARACTERISTICS.
AND WITH THE SHARED
CHARACTERISTICS WE CAN
POTENTIALLY FIND SHARED TARGETS
AND SHARED INTERVENTION.
RATHER THAN LOOKING AT ONE
DISEASE AT A TIME WE WANT TO
LOOK AT CLUSTERS AT A TIME.
HOW DO WE CLUSTER THEM ?
WE PUT DIFFERENT KINDS OF
INFORMATION TOGETHER IN THIS
CASE WE HAVE INFORMATION THAT
COMES FROM OUR OWN RESOURCES BUT
IT'S ALSO SUPPLEMENTED BY
OUTSIDE RESOURCES SUCH AS
PATHWAY COMMONS AND CREATE AN
INTEGRATED NETWORK.
THIS REFLECTS MOLECULAR
MECHANISMS AND ALSO VARIOUS
TYPES OF PHENOTYPES.
SO WE EMPLOYED A NATURAL
LANGUAGE ALGORITHM TO DEFINE
CLUSTERS YOU CAN SEE ON THE Y
AXIS SO EACH ROW HERE SAY
CLUSTER AND ON THE X AXIS
THERE'S CHARACTERISTIC THAT ARE
DRAWN.
IF YOU ZOOM INTO ONE ROW HERE
YOU CAN SEE THEY'RE SHARING THE
RED AND BLUE COLOR AND THOSE
MEASURE THE IMPACT OF THOSE
SPECIFIC CHARACTERISTICS.
THE CHARACTERISTICS ON MOLECULAR
MECHANISM OR DIFFERENT TYPES OF
PHENOTYPES.
AS AN EXAMPLE I WANT TO
HIGHLIGHT ONE OF THE CLUSTERS.
THE CLUSTER CONTAINS
CLARCOT-MARIE TOOTH AND
METATROPIC DYSPLASIA AND THEY
SHARE KEY CHARACTERISTICS AND I
LISTED SOME HERE INCLUDING
MUSCLE FILL AMOUNT SLIDING OR
MUSCLE ORGAN DEVELOPMENT AND
ORGANIZATION.
THIS IS IMPORTANT BECAUSE NOW WE
HAVE THE SHARED CHARACTERISTICS
AND WE CAN START LOOKING FOR
APPROPRIATE WAYS OF INTERVENING
INTO THE PATHWAYS AND TARGETING
THEM.
THIS IS THE NEXT STEP.
SO THIS WORK IS LAYING A
FOUNDATION FOR MANY FUTURE WORK
SO STAY TUNED.
ANOTHER EXAMPLE I'D LIKE TO
HIGHLIGHT IS THE NCATS
TRANSLATOR.
THIS IS A LARGE EFFORT THAT IS
LED BY THE EXTRAMURAL PROGRAM AT
NCATS AND AGGREGATES INFORMATION
ACROSS THE SPECTRUM FROM BASIC
TO RESEARCH TO CLINICAL.
AND THIS IS A LARGE
COLLABORATIVE EFFORT.
WE'RE INVOLVED AND MANY NUMEROUS
ACADEMIC INSTITUTIONS ARE AS
WELL.
AND VERY RECENTLY WE SEE HERE ON
THE RIGHT AN EXAMPLE OF THE
ASSAY AND YOU CAN QUERY
TRANSLATOR.
SO THERE'S SPECIFIC TYPES OF
QUERIES YOU CAN MAKE.
ONE I'D MAKE HERE IS ON GLIAL
BLASTOMA AND WE'RE LOOKING FOR
PUNITIVE TARGETS OF GLIAL
BLASTOMA AND WANTED TO COMPARE
THE TRANSLATOR.
AND WHEN I LOOK HERE FOR ONE
DRUG MAY TREAT GLIAL BLASTOMA,
WHAT GETS RETURNED IS NOT ONLY A
LIST OF DRUGS BUT ALSO TELLS YOU
WHY IT FOUND THOSE PARTICULAR
DRUGS.
SO THE FIRST HIT HERE IS IF YOU
LOOK HERE ON THE BOTTOM YOU CAN
SEE THAT THE DRUG IN YELLOW
LINKED TO THE DISEASE GLIAL
BLASTOMA IN BLUE AND WHY THEY'RE
CONNECTED.
AND WHAT'S MOST INTERESTING
ARGUABLY HERE IS THE
RELATIONSHIP WITH THE KINASE
WHICH IT TARGETS AND THIS IS
ASSOCIATED WITH GLIAL BLASTOMA
AND SOMETHING WE FOUND IN OUR
OTHER STUDY IN THE SIMILAR
INDIRECT WAY AND LOOKING AT
CLINICAL TRIALS THERE'S A HUGE
CLINICAL TRIALS EVALUATING FOR
THE GLIAL BLASTOMA.
THIS IS HOW WE FIT THIS TYPE OF
INFORMATION.
ONE ASPECT IS TRUSTWORTHINESS IN
THE RESULTS.
WITH CHAT GPT AND THE SOFTWARE
THAT ARE REALLY AT OUR FINGER
TIPS DON'T REALLY PROVIDE
INFORMATION HOW THEY GOT TO
THEIR ANSWERS.
BUT YOU HAVE TO MAKE SURE THE
ANSWERS ARE THEMSELVES
TRUSTWORTHY AND THERE'S
TRANSPARENCY IN THAT THROUGH
TRANSLATOR AND ALSO THROUGH THE
KNOWLEDGE GRAPHS WE BUILD IN
GENERAL AND I THINK IT'S A VERY
KEY POINT.
LASTLY I WANT TO TALK A LITTLE
BIT ABOUT NEW TECHNOLOGIES WE'RE
BRINGING HERE AT NCATS
SPECIFICALLY RELATED TO OMICS.
AND SO THE OMICS IS VERY
IMPORTANT BECAUSE IT ENABLES THE
COMPREHENSIVE PROFILING OF
METABOLITES, GENES AND PROTEINS
AND THOSE ARE IMPORTANT FOR
DISCOVERING BIOMARKERS AND
IDENTIFYING THE MECHANISMS THAT
UNDER LIE DISEASES AND DRUG
RESPONSE.
AND SO WE'RE VERY INVOLVED THE
ROBUST DATA GENERATION INCLUDING
STUDY DESIGN AND WE'RE ALSO
DEVELOPING RECRUITING PROTOCOLS
AT SCALE AND WE'RE DOING THIS
WITH METABOLOMICS.
THIS IS AN EXAMPLE OF A RECENT
INSTRUMENT THAT WE'VE ACQUIRED
WITH OUR IN-HOUSE COLLABORATORS
IN ANALYTICAL CHEMISTRY.
THE REASON TO DO THIS IS BECAUSE
NCATS TENDS TO DO THINGS AT
SCALE AND VERY COSTLY TO DO THIS
AT SCALE IF WE SHIP OUT THE
SAMPLES FOR THAT ANALYSIS.
SO WE WANT TO BRING THIS
IN-HOUSE TO BE ABLE TO DO IT.
AND THEN IN PARALLEL, WE'RE ABLE
TO ALSO PRODUCE THE ROBUST DATA
ANALYTICS AND THIS INCLUDES
NOVEL METHODOLOGY BUT THOSE TWO
GO HAND IN HAND AND THIS IS A
TREND.
PEOPLE THINK OF THE DATA
GENERATION AND GET TO A POINT
WHERE THERE'S A LOT OF DATA AND
WHAT ARE WE GOING TO DO WITH IT
AND BUILD THE INFRASTRUCTURE.
HERE WE'RE SPOILED IN THAT WE'RE
ABLE TO DO BOTH IN PARALLEL AT
THE SAME TIME.
WE'RE SETTING OURSELVES UP TO BE
ABLE TO HANDLE THE BIG DATA ONCE
IT COMES AND WE'RE ALSO MAKING
SURE THAT ALL THESE ANALYTICS
ARE INTEROPERABLE WITH OTHER
DATA TYPES BEING GENERATED AT
NCATS INCLUDING THE IMAGING
DATA.
WE'RE ALL IN COMMUNICATION TO BE
AS
ANOTHER WORK WE'RE DOING TO
HOUSE MOLECULAR DATA UNDER ONE
SYSTEM TO FACILITATE THE REUSE.
RIGHT NOW THERE'S NO PUBLIC
MULTIOMIC REPOSITORY THAT EXISTS
THAT INCLUDES THE ANALYTICS, SO
YOU MAY BE ABLE TO GET ACCESS TO
DATA, BUT YOU STILL HAVE TO DO
THE WORK TO PUT IT TOGETHER.
AND YOU STILL HAVE TO HAVE THE
ANALYTICS AND THAT KNOWLEDGE IN
HOUSE.
WE HAVE A COUPLE GUIDING
PRINCIPLES THAT WE'RE APPLYING
AS WE'RE DEVELOPING THIS.
WE WANT TO USE STANDARD DATA
FORMATS, AND STANDARD
PRE-PROCESSING.
WE WANT A DIRECT ACCESS TO
ANALYTICS TO ANALYZE THE DATA.
WE WANT IT TO BE INTEROPERABLE
WITH OTHER NCATS RESOURCES.
BOTTOM LEFT, AGAIN, BEING ABLE
TO LOOK AT THAT MEDIA GENERATED
IN LIGHT OF THE PREVIOUS
KNOWLEDGE FROM THE KNOWLEDGE
SOURCES, THAT WE'VE BEEN
BUILDING.
HERE THE SCIENTIFIC GOAL IS
BRIDGE THE GAP BETWEEN
PRE-CLINICAL AND CLINICAL
EFFORT, TRY TO DERIVE ACTIONABLE
INSIGHTS FROM THE DATA.
ON THE TOP RIGHT THIS IS JUST
OUR INTERFACE RIGHT NOW, IT'S IN
DEVELOPMENT.
IT MAY CHANGE.
SO, OVERALL, YOU KNOW, IT'S
ABOUT TURNING DATA INTO
ACTIONABLE INSIGHT, ACCELERATING
TRANSLATION AND FINDINGS TO THE
CLINIC.
A LOT OF DATA IS COMING OUR WAY,
INTERPRETATION IS VERY
DIFFICULT, BUT WE'RE PUTTING THE
TOOLS TOGETHER TO BE ABLE TO
MAKE THAT INTERPRETATION
EFFICIENT.
AND ALSO ABLE TO MAKE IT AS
ROBUST AS POSSIBLE.
WE'RE REALLY ABLE TO DO THIS
THROUGH TEAM SCIENCE CULTURE, WE
HAVE THE CULTURE BUT IT
PERMEATES THROUGHOUT NCATS.
AND THAT MULTI-LINGUAL ASPECT OF
OUR TEAM THAT WE'RE ABLE TO TOSS
TO DIFFERENT EXPERTS, CHEMISTRY,
BIOLOGY, ET CETERA.
WE HAVE A LOT OF COLLABORATIONS
WITHIN NCATS, MORE BROADLY AT
THE NIH.
MANY REGULATORY AGENCIES,
ACADEMIA, ALSO SOME IN INDUSTRY.
AND AT THE END OF THE DAY WE
WANT TO DERIVE ACTIONABLE
INSIGHTS THROUGH DATA
INTEGRATION AND BUILD ON WHAT WE
KNOW TO ENHANCE TRANSLATIONAL
SCIENCE EFFORTS.
SO, WITH THAT I HOPE YOU'RE AS
EXCITED AS I AM, AND OUR EFFORT
TO REALLY TRY TO MAKE THE VISION
A REALITY, PROVIDING MORE
TREATMENT TO MORE PATIENTS.
THIS IS OUR GROUP.
WE MET FOR THE FIRST TIME LAST
FALL.
SO, AS ANTON MENTIONED, WHEN I
CAME IN I ONLY HAD A MONTH AND A
HALF, A LOT OF PEOPLE I MET FOR
THE FIRST TIME LAST FALL, A BIT
FUNNY.
AND THANK YOU FOR YOUR
ATTENTION.
[APPLAUSE]
>> THANK YOU, EWY.
I WOULD LIKE TO SEE IF THERE ARE
ANY QUESTIONS FOR EWY AROUND THE
TABLE.
>> I DON'T HAVE A QUESTION.
I HAVE A COMMENT.
INFORMATICS IS NOT MY
WHEELHOUSE, BUT POLICY IS.
BEING AN END USER IN THE
COMMUNITY I CAN JUST SAY WE'VE
BEEN TALKING, WHEN WE DID THE
LAST TOUR, COVID LEARNINGS, HOW
WE'RE PULLING THEM BACK.
AND ONE OF THE THINGS IN THE
COMMUNITY FOR A VERY LONG TIME
WE'VE BEEN WANTING AND PUSHING
FOR, REAL WORLD DATA TO BE ABLE
TO INFORM DIFFERENT AREAS OF THE
ECOSYSTEM, ESPECIALLY LABEL
EXPANSION.
AND SO JUST SEEING HOW THAT
TECHNOLOGY IS BEING UTILIZED
HERE TO DO THAT, HAPPENING
WITHIN COVID BUT APPLICATIONS
BEYOND THAT, IT'S REALLY MUSIC
TO OUR EARS.
LANGUAGE IN THE 21ST CENTURY
CURES ACT, GUIDANCES HAVE COME
OUT OF FDA SHOWING THAT CAN BE
DONE BUT SEEING THAT PUT IN USE
WHEN NCATS AND FDA, CRITICAL
PATHS, IT'S REALLY WONDERFUL.
WE NEED MORE BUT TO SEE NCATS AT
THE CENTER AND HELPING DRIVE
THAT IS CRITICALLY IMPORTANT.
I JUST WANTED TO FLAG THAT AND
THANK YOU FOR THAT, PUSH
LANGUAGE INTO FEDORA TO MAKE
SURE EVERYONE THAT NEEDS TO HAS
EXPLICIT AUTHORITY TO DO THAT.
THANK YOU FOR YOUR WORK ON THAT.
>> IN A SHORT AMOUNT OF TIME,
THE PROGRESS, BUT A QUESTION IS,
WHAT IS THE PROTOCOL FOR THE
SCIENTIFIC COMMUNITY, WONDERFUL
CONTRIBUTION, LOTS OF PEOPLE
COULD BENEFIT FROM.
>> YEAH, SO RIGHT NOW IT'S
INTERNAL ONLY.
HOWEVER, THERE'S NO REASON WHY
IT COULD BE PUBLICLY RELEASED,
AND PROBABLY, YOU KNOW, ONE
MODEL THAT WE'VE BEEN THINKING
ABOUT IS SIMILAR TO THE GSRS
MODEL, WHERE YOU HAVE AN
INSTANCE OF THE SOFTWARE AT THE
FDA AND ANOTHER AT AREA
INSTITUTIONS, IT CHANGES
ACCORDING TO THEIR ENVIRONMENT,
AND THAT'S WHAT WE'RE THINKING
ABOUT.
BUT INITIALLY IT'S GOING TO BE
IN HOUSE.
BUT, YES, THAT'S WHERE WE'RE AT
RIGHT NOW.
>> MATTIAS?
>> ALWAYS GREAT TO SEE A
PARTNER BUILDING INFORMATICS
INFRASTRUCTURE FOR THE NEEDS OUT
THERE.
OBVIOUSLY, YOU HAVE A VERY
POWERFUL AND EFFECTIVE TEAM.
BUT NEVER INFORM INFORMATICIANS,
SOFTWARE DESIGNERS, PROJECT
MANAGERS TO DO THAT.
WHAT'S YOUR PROCESS TO IDENTIFY
AND PRIORITIZE TEAM BUILDING AND
AFTER YOU BUILD IT AND OBVIOUSLY
THE DESIGN PRINCIPLES, AFTER YOU
BUILD IT, HOW DO YOU MAINTAIN IT
AND KEEP IT ROBUST AND RESILIENT
TO THE USER?
>>I MEAN, YOU KNOW, THESE ARE
THE MILLION DOLLAR QUESTIONS,
RIGHT?
TRYING TO FIGURE OUT WHAT TO
PRIORITIZE AND WHAT TO MAINTAIN.
SO IN TERMS OF PRIORITIZATION, I
THINK THERE'S -- SOME OF THESE
PROJECTS ARE DRIVEN THROUGH
EXTRAMURAL FUNDS, AND SO THAT'S
HOW WE CAN KEEP DEVELOPING
THOSE.
OBVIOUSLY WHEN THEY REACH SUCH A
BROAD IMPASSE, ESPECIALLY
INTERNATIONALLY, THOSE ARE
DEFINITELY PROJECTS THAT WE WANT
TO, YOU KNOW, KEEP WORKING ON.
ON THE FLIP SIDE, YOU HAVE TO
HAVE THESE FRESH NEW IDEAS,
RIGHT?
ALWAYS NEW DATA IS BEING
GENERATED.
AND SO WE HAVE A NUMBER OF
TRAINEES THAT ARE DEVELOPING
KIND OF THESE NEW METHODS,
LOOKING AT NEW TECHNOLOGIES,
BRINGING IN THOSE FRESH IDEAS,
AND SOME OF THEM JUST STICK.
AND THEY STICK BECAUSE WHEN WE
TALK ABOUT THEM, THEN OTHERS
FIND USE CASES FOR THEM, RIGHT?
AND I THINK THE USE CASE IS
WHAT'S DRIVING OUR
PRIORITIZATION.
IF THERE'S MULTIPLE PEOPLE THAT
COME IN AND WE'RE LIKE, OH, THIS
IS SUPER USEFUL, MAYBE YOU CAN
ADD THIS FEATURE, THEN, YEAH, I
MEAN, WE'RE GOING TO PRIORITIZE
IT AND GET IT DONE BECAUSE
THAT'S HOW -- IT BECOMES A
SNOWBALL EFFECT, RIGHT?
WHEN USAGE GOES DOWN OR USE
CASES BECOME OBSOLETE THAT'S
WHEN WE DEPRIORITIZE.
THIS IS ALWAYS A WORK IN
PROGRESS.
NOBODY LIKES TO KILL PROJECTS.
WE HAVE WORKED TO KILL SOME
PROMISE JUST TO ADVANTAGE
BANDWIDTH BUT THIS IS AN ONGOING
CHALLENGE.
MAYBE IT'S A REALLY HARD ONE.
YOU KNOW, IT'S USUALLY EASIER TO
GET EXCITEMENT AND SUPPORT,
MEANING BUDGET SUPPORT IN THIS
CASE, FOR BRAND NEW SHINY
THINGS.
NOBODY REALLY WANTS TO FUND
THINGS THAT HAVE BEEN AROUND.
IT JUST GETS KIND OF PUSHED TO
THE NEXT PERSON, YOU KNOW, IN A
WAY.
AND THAT'S NOT A FAULT.
THAT'S JUST I GUESS MY
OBSERVATION.
AND IT'S HARD, PEOPLE GET USED
TO HAVING THOSE RESOURCES, IT
DOESN'T WORK, FOR ME, THAT'S THE
END OF THE WORLD.
THERE'S A HIGH EXPECTATION OF
WORKING THERE AND BEING THERE,
RIGHT?
AND SO ONE OF THE WAYS THAT
WE'RE THINKING ABOUT DOING THIS
IS BASICALLY TO BUILD USER
COMMUNITIES AND TO GET BROADER
ADOPTION OUTSIDE OF OUR
IMMEDIATE GROUPS.
THIS OF COURSE REQUIRES EFFORT,
BECAUSE THEN FAMILIARIZE PEOPLE,
ET CETERA, THIS IS HOW WE'RE
THINKING ABOUT IT, AND ALSO
ENGAGING WITH OTHERS.
OTHER INSTITUTIONS HAVE STRONG
USE CASES, THEN, YOU KNOW, WE
CAN GET SOME SUPPORT FROM THEM
AS WELL.
>> THANKS A LOT.
OBVIOUSLY IT'S A EFFICIENT WAY,
I PARTICULARLY LIKE THE
BOTTOM-UP APPROACH, YOUR TEAM
MEMBERS GET EXCITE AND HAVE
INSIGHT TO SCALE THEM UP TO
SOLUTIONS WHICH ARE NOT LAB.
IT ALSO BEHOOVES YOU TO IDENTIFY
WHAT ARE CRITICAL AREAS UNIQUE,
WHAT SETS YOU APART FROM LARGE
INFORMIC POOL OUTSIDE THE NCATS
PROGRAM AND THAT'S WHAT I WOULD
EXPECT TO SEE PROGRAMMATIC
STRATEGIC PLAN TO SAY THOSE ARE
AREAS WHERE ONLY WE CAN MAKE THE
DIFFERENCE, AND NOT, YOU KNOW,
THE GROUP IN HARVARD GENERATING
DATA, WHICH THEY PROBABLY COULD
HAVE BUILT THESE TOOLS AS WELL.
BUT WHAT MAKES YOU UNIQUE AND
THEREFORE ONLY YOU CAN DO THAT?
THOSE SHOULD BE PROJECTS TO GET
EXTRA PRIORITY STAR ATTACHED TO
THEM.
>> GREAT POINT.
THANKS, MATTIAS.
PAUL HARRIS, DID I SEE YOUR HAD
UP?
>>YES.
IMPRESSIVE PORTFOLIO, WHAT
YOU'VE DONE IN A SHORT AMOUNT OF
TIME.
QUICK QUESTION, GOING BACK TO
USE CASE WHERE YOU WERE
COLLABORATING WITH HARVARD, WAS
THAT TIED TO MPC OR A DIFFERENT
PROJECT?
>> IT WASN'T.
VERY INTERESTING QUESTION.
I WOULD LOVE, LOVE TO TIE THAT
DATA WITH N3C, BECAUSE ONE OF
THE ASPECTS, ONE OF THE
STRENGTHS OF N3C IS THAT THERE'S
A LOT OF PHENOTYPES AVAILABLE AT
YOUR FINGERTIPS, BECAUSE INSIDE
THE ENCLAVE YOU GET ACCESS TO
THE CODE AND OTHER PEOPLE'S
WORK.
IF YOU'RE LOOKING FOR A CERTAIN
PHENOTYPE YOU CAN LITERALLY GRAB
IT OR GRAB THE CODE AND APPLY TO
YOUR COHORT.
TO DO THAT, OUTSIDE OF N3C, IF
YOU THINK OF THE THOUSANDS OF
PHENOTYPES, IT'S NOT POSSIBLE,
RIGHT?
IT'S REALLY, REALLY HARD TO DO.
AND SO I'M STILL AT IT.
I HOPE THAT WE WILL BE ABLE TO
MAKE THAT LANGUAGE.
I THINK IT'S ONE OF THOSE THINGS
TECHNICALLY IT'S POSSIBLE,
AGREEMENT-WISE IT GETS
COMPLICATED.
THAT'S A GREAT QUESTION.
YEAH, I REALLY HOPE WE CAN DO
THAT.
ALL THIS WORK, THERE'S A LOT OF
WORK THAT'S BEEN GOING ON FOR
MANY, MANY YEARS PRIOR TO ME.
I HAD THE LUXURY OF COMING IN TO
SOME ALREADY GREAT WORK, AND I
THINK IT WAS A MATTER OF KIND OF
CONSOLIDATING AND THINGS OF THAT
SORT.
I WANTED TO EXPLICITLY THANK
YOU, BUT I WANTED TO MENTION
THAT.
IT'S A GREAT TEAM.
>> THANK YOU.
TO THAT POINT, YOU HIT THE
GROUND RUNNING DESPITE THE FACT
HAD YOU A PANDEMIC IN THE
BEGINNING OF YOUR TENURE YEAR
AND REALLY MADE SUCH HEADWAY.
AND ALSO JUST TO THE POINTS,
MANY OF THE POINTS THAT EWY
MENTIONED, THINKING ABOUT THE
DATA SCIENCE, INFORMATICS,
INTEROPERABILITY, EXPLICIT
STREAMS OF WHAT WE'RE DOING IN
THE STRATEGIC PLAN ALSO, AND
YOU'RE NOT HEARING AS MUCH ABOUT
THAT BECAUSE IT'S MORE OF A
DEEPER DIVE.
BUT WE ARE REALLY LOOKING INTO
HOW THIS IS FOUNDATIONAL TO A
LOT OF WORK WE'RE DOING, A PART
OF WEAVING THAT INTO THE FABRIC
OF WHAT IS NCATS AND WHAT WILL
MAKE UP THE STRATEGIC PLAN.
YOU'LL BE HEARING AND SEEING A
LOT OF THE FINGERPRINTS THAT
WERE TALKED ABOUT TODAY AS WELL
AS WE PROCEED IN THAT PROCESS.
AMIKA, YOUR HAND IS UP.
GO AHEAD.
>> QUICKLY, TO UNDERSCORE THE
IMPACT THIS WORK CAN HAVE WHEN
YOU'RE TALKING ABOUT MAINTENANCE
AND, YOU KNOW, ELEVATING NCATS
IN TERMS OF IMPACT, BECAUSE I
THINK WHAT YOU'VE SHOWN IS HOW
YOU CAN TAKE INFORMATICS AND
THESE DATABASES ARE MAKING IT
MORE USER FRIENDLY, WHICH WILL
CREATE MORE USERS OF THE
DATABASE, SO I THINK THAT, FOR
ONE, IS SOMETHING TO CONTINUE TO
PUSH, HOW TO CREATE OUTSIDE OF
THE INFORMATICS WORLD THAT CAN
NOW USE THIS.
AND THEN SECONDLY GOING BACK TO
THE POLICY PIECE BECAUSE I THINK
THAT N3C BE STORY THAT JONI
TOLD, YOU KNOW, I'M REALLY
INTERESTED TO SEE WHERE THAT
GOES BECAUSE IT SEEMS LIKE
THAT'S A REALLY COMPELLING PIECE
OF EVIDENCE THAT WILL HELP
POLICIES OR NEW MEMORIAL
PRACTICES NOW BECOME, BECAUSE OF
THAT WORK.
SO I THINK CONTINUING TO TELL
THE STORY, MORE USERS OUTSIDE OF
INFORMATICS AND POLICIES,
PRACTICES THAT CAN NOW BE
CREATED BECAUSE OF THIS WORK.
>> THANK YOU FOR THAT COMMENT.
THAT'S EXACTLY WHERE WE WANT IT
TO GO.
THAT'S GREAT.
WE ARE AT TIME, WE'RE GOING TO
MOVE TO THE NEXT PRESENTATION.
SO I WANT TO SAY THANK YOU TO
EWY MATHE AND ANTON, FOR HIS
WORK AND ROLE AS THE SCIENTIFIC
DIRECTOR IN DPI, AND REALLY
PROVIDING THE ABILITY FOR THE
SCIENTISTS WITHIN DPI TO GIVE
YOU A DEEPER DIVE IN TERMS OF
WHAT WE'RE DOING.
THE WORK IS VAST AND IT'S
HELPFUL TO GET THAT BIGGER
PICTURE.
SPEAKING OF A BIGGER PICTURE IN
TERMS OF RARE DISEASES, WE WILL
TURN IT OVER NOW TO P.J. BROOKS,
ACTING DIRECTOR OF DRDRI.
HE'S GOING TO INTRODUCE PROBABLY
TIIANA URV, PROGRAM DIRECTOR.
P.J., OVER TO YOU.
>> THANKS, JONI.
HAPPY TO PRESENT ON BEEHIVE OF
DRD RISKERI MANY ACTIVITIES
GOING ON, I WON'T BE ABLE TO
TALK ABOUT ALL, I'LL HIT SOME
HIGHLIGHTS.
BUT THE BIGGEST ONE IS THE RARE
DISEASE RESEARCH NETWORK.
I WANT TO GIVE TIINA A CHANCE TO
TALK ABOUT THAT AND FOLLOW UP
WITH OTHER HIGHLIGHTS.
>> I'M HERE TO TELL YOU ABOUT
THE CLINICAL RESEARCH NETWORK.
RDCRN WAS ESTABLISHED IN 2002,
PROGRAM HAS BEEN AROUND FOR SOME
TIME.
NIH WAS DIRECTED TO ESTABLISHING
REGIONAL CENTERS OF EXCELLENCE,
FIRST FUNDED IN 2003, HAVE GONE
CONSISTENTLY THROUGH COMPETITION
EVERY FIVE YEARS.
NEXT SLIDE.
THE CURRENT RDCRN IN A NETWORK
OF 20 CONSORTIA OR A NETWORK OF
NETWORKS, BUT THE DATA
MANAGEMENT AND COORDINATED
CENTERS.
NCATS PARTNERED WITH TEN
INSTITUTIONS AND CENTERS FOR THE
PROGRAM LISTED HERE.
THE NIH MECHANISM USED TO
SUPPORT THE NETWORK IS U54
COOPERATIVE AGREEMENT, THERE'S
SIGNIFICANT PROGRAMMATIC
INVOLVEMENT FROM NIH PROGRAM
OFFICIALS AND THERE'S A LOT OF
PARTNERSHIP AND INTERACTION
BETWEEN PROGRAM OFFICERS AND
SCIENTIFIC OFFICERS AND
GRANTEES, AND THE PATIENT
ADVOCACY GROUPS WHICH ARE AN
IMPORTANT PART OF DISCOVERY.
NEXT SLIDE PLEASE.
SO THE FOCUS OF THE NETWORK
RIGHT NOW IS CLINICAL TRIAL
READINESS.
STUDIES THAT MOVE THE CLINICAL
GROUP CLOSER TO SUCCESSFUL
CLINICAL TRIALS, FOR EXAMPLE
NATURAL HISTORY STUDIES, OR THE
IDENTIFICATION OF BIOMARKERS AND
DEVELOPMENT OF ENDPOINTS
APPROPRIATE FOR CLINICAL TRIALS.
EACH INDIVIDUAL CONSORTIUM
STUDIED THREE OR MORE DISORDERS,
CONSISTS OF MULTIPLE SITES, AND
THREE TO FIVE CLINICAL STUDIES,
CLINICAL PROGRAM FOR STUDIES,
CAREER ENHANCEMENT PROGRAM THAT
THEY DESIGN THEMSELVES AND HAVE
FULLY INTEGRATED PATIENT
ADVOCACY.
DATA MANAGEMENT AND COORDINATING
CENTER PROVIDES SUPPORT TO BOTH
INDIVIDUAL CONSORTIUM AND
NETWORK.
DMC PROVIDES ADMINISTRATIVE
SUPPORT, OPERATIONS, GOVERNMENT
CLINICAL RESEARCH SUPPORT THAT
COORDINATES BEST PRACTICES,
STANDARDIZATION, DATA MANAGEMENT
IN COOPERATION WITH NCATS ITRB
BUILDS AND MAINTAINS SECURE DATA
INFRASTRUCTURE.
AND ENGAGEMENT AND DISSEMINATION
FOR SUPPORT, PATIENT ENGAGEMENT
AND BROAD OUTREACH FOR
DISSEMINATION.
NEXT.
THERE'S A BROAD REACH.
WE HAVE 358 ACTIVE CONSORTIA,
197 ARE AT UNIQUE LOCATIONS
WHICH MEANS, FOR EXAMPLE, NINE
CONDUCTING CARE DISEASE RESEARCH
AS PART OF THE RDCRN AT THAT
SITE AND PARTNER
INTERNATIONALLY.
WE HAVE 11 COUNTRIES HAVING
ACTIVE RESEARCH SITES ACROSS THE
NETWORK.
RDCRN IS CO-LOCATED WITH OUR
CTSAs.
SO, LEADERSHIP HAS THREE
BRANCHES.
P.I. LEADERSHIP TEAM.
THE PATIENT ADVOCACY LEADERSHIP
TEAM.
AND THE EXECUTIVE LEADERSHIP
TEAM WHICH IS MADE OF BOTH THE
CPAG AND P.I., GUIDED BY
REPRESENTATIVES AND PATIENT
ADVOCACY GROUP.
THIS GROUP COORDINATED WITH THE
NIH REPRESENTATIVE.
ELECTED BY PEERS, SCHEDULED
ROTATION, THE VETTING BODY FOR
ACTIVITIES WITHIN THE NETWORK
WITH A WIDE VARIETY OF INTEREST
GROUPS WITH FOCUS ON RESEARCH.
ONE OF THE THINGS THAT WE REALLY
LIKE ABOUT THE NETWORK IS THESE
WORKING GROUPS ARE OPEN TO
ANYONE.
IT'S NOT JUST THE PRINCIPAL
INVESTIGATORS ON IT OR
CO-P.I.s, IT'S ANYONE WHO
SHOWS INTEREST, BIOINFORMATICS,
ANYONE.
SO THE PROGRAM HAS BEEN AROUND
FOR ALMOST 20 YEARS.
WE LIKE TO KEEP IT FRESH AN KEEP
UP WITH COMPLIANCE AND LOOK AT
WHERE THE NEEDS ARE IN THE RARE
DISEASE COMMUNITY.
WE'RE PREPARING TO GO INTO CYCLE
4, RDCRN-4.
ONE THING WE DID WAS EVALUATE
PROGRAMS, DATA WAS PRIMARY
IMPORTANCE.
SHARING TOOLS AND ENVIRONMENT,
NOT JUST LOOKING AT TIMES, WE'RE
PROVIDING THE TOOLS AN RESOURCES
TO THE WHOLE NETWORK AT ONE
TIME.
INSTEAD OF GETTING A GRANT,
PAYING FOR LICENSES, FILE
STORAGE, MONEY GOES TO RESEARCH,
NCATS PICKS UP THE TAB FOR THE
DIFFERENT COURSES.
WE HAVE TWO ENVIRONMENTS.
FIRST IS OPERATIONAL
ENVIRONMENT, THE WORKING
ENVIRONMENT, DEVELOPED FOR THE
CONSORTIA RESEARCH.
IT'S AN INTERNAL SITE WHERE THE
TOOLS ARE AVAILABLE AND SHARED
ACROSS THE CONSORTIA.
TOOLS NOW INCLUDE REDCap.
UM BRA, JUPITER HUB, NIH
TOOLBOX, THERE'S A VARIETY.
THE NUMBER KEEPS GROWING.
THIS LIVES AT NCATS BUT IS
MANAGED BY OUR DATA MANAGEMENT
COORDINATING CENTER.
THE ENVIRONMENT WAS DEVELOPED
THROUGH CLOSE COLLABORATION,
COLLABORATIVE RELATIONSHIP, ALL
THE COSTS WHICH WE HOPE TO
CONTINUE DOING.
WE MANAGE THE SECURITY.
WE HAVE MULTI-FACTOR
AUTHENTICATION THROUGH DUO, SO
THERE'S NO ON-SITE
INFRASTRUCTURE.
THANK YOU FOR COMING.
AND ALL OF THIS IS RUNNING ON
AWS, THIRD PARTY CONTRACTS,
OTHERWISE HELD BY NCATS.
NEXT SLIDE PLEASE.
WE WERE THINKING IF WE BUILD IT,
WILL THEY COME?
WE DID THIS.
AND THEY ARE USING.
MUCH CAN BE ATTRIBUTED TO
REACHING OUT TO INVESTIGATORS
FINDING OUT WHAT TYPES OF TOOLS
THEY NEEDED AND GIVING
OPPORTUNITY TO TRY THEM OUT IN A
SANDBOX ENVIRONMENT.
NEXT SLIDE.
BENEFIT OF WORKING AT NIH YOU
KNOW WHAT'S COMING DOWN THE
PIKE.
WE BEGAN PREPARING FOR IT EARLY.
IN FACT, WHEN WE WERE DEVELOPING
THE RFA OF THE CYCLE WE KNEW
THEY HAD DONE THAT DOWN THE
LINE.
WHAT THAT MEANS FOR THE RDCRN IS
THAT WE HAVE A DEVELOPMENT
REQUIREMENT, AND THIS PORTION OF
THE PROGRAM IS PERSONALLY BEING
BUILT, TAKES CONSENSUS DATA, DEI
WAS IT, DMCC WILL PROVIDE
ASSURANCES OF DATA QUALITY.
ONE OF THE THINGS OUR DNC DOES
IS LOOK TO OTHER RARE DISEASE
GROUPS COLLECTING DATA.
AND A LOT OF DATA STANDARDS.
WHILE WE KNOW EVERYONE WILL
AGREE WITH ONE SET OF DATA
STANDARDS WE WANT TO REMAIN
INTEROPERABLE SO WHEN THEY HAVE
ACCESS THEY WILL BE ABLE TO
COMBINE THE DATA.
THE ENVIRONMENT, ONCE THE DATA
IS HANDED OFF TO THE SHARED
ENVIRONMENT, THIS ENVIRONMENT
THAT IS GOVERNED WILL BE
CONTROLLED AND WILL HAVE
CONTROLLED DATA ACCESS TO NCATS
DECKS.
IT IS THE INTENT THIS WILL BE
AVAILABLE FOR THE PUBLIC THROUGH
CONTROLLED ACCESS.
SO HOW ARE WE DOING, FOR QUITE
SOME TIME, NIH, I'D LIKE TO
THANK JOANNE, WHO I WORKED
CLOSELY TO WHO DID THESE
ANALYSES FOR US.
IDENTIFIED BY HUB, 28
CONSORTIUMS, THERE HAVE BEEN
2,
2700 PAPERS.
THE SAME DATA WAS USED TO
IDENTIFY ORGANIZATIONS THAT
PUBLISHED AT LEAST FIVE PIPES.
SO REACH OF THE NETWORK HAS BEEN
EXPANDING, NUMBER OF
ORGANIZATIONS INCREASE THE
GREATLY OVER TIME.
THERE'S A BIG INCREASE IN NUMBER
OF INTERNATIONAL COLLABORATIONS,
WHICH YOU CAN SEE ESPECIALLY IN
2020, 50% OF OUR COLLABORATORS.
WE LOOK AT CO-SHORESHIP.
WHAT YOU SEE -- CO-AUTHORSHIP.
DESPITE THE INCREASE IN SIZE,
NUMBER OF CLUSTERS IS DIFFERENT
GROUP, THINK OF THEM AS PERHAPS
DIFFERENT CONSORTIA.
THAT FLINT -- THAT DIDN'T CHAN.
MORE PEOPLE ARE JOINING,
OUTREACH IS GROWING.
LET'S LOOK AT THE IMPACT OF
NETWORK ON TRANSLATIONAL
OUTCOMES.
WE CAN PUT CLINICAL TRIALS INTO
TWO CATEGORIES, FUNDED BY U54
AND THOSE NOT FUNDED DIRECTLY,
BUT LEVERAGE RDCRN RESOURCES.
THE DIRECTLY FUNDED TRIALS ARE
SMALL PHASE 1 AND PHASE 2
CLINICAL TRIALS, 8 CLINICAL
TRIALS IN TOTAL SINCE 2003
CURRENTLY WITH 18 ONGOING
TRIALS.
22 OF THE 33 CONDUCTED AT LEAST
ONE CLINICAL TRIAL.
YOU'LL SEE THE RDCRN ASSOCIATED
TRIALS HAS BEEN PHASE 2, PHASE
3, AND AT LEAST 13 ONGOING
CLINICAL TRIALS THAT FALL INTO
THE CATEGORY AT THIS TIME.
THAT FUNDING COMES FROM
INDUSTRY, WE HAVE BUILT A
FOUNDATION OF CLINICAL
RESEARCHERS, NATURAL HISTORY, WE
HAVE ALL KINDS OF INFORMATION
FOR THEM TO USE.
SO THE OTHER FUNDING HAS COME
FROM INDUSTRY, SPECIFIC GRANTS,
FDA AND PATIENT ADVOCACY GROUPS.
THEY HAVE LEVERAGED PATIENT
POPULATION, CLINICAL ENDPOINTS,
BIOMARKERS, SAFETY AND EFFICACY
DATA.
TO DATE RDCRN HAS 8 APPROVED
TREATMENTS FOR RARE DISEASE.
JONI THOUGHT THE PIPELINE AND
THEN WE'VE TAKEN ADVANTAGE OF
HOPEFULLY HAPPY WE'RE EMBRACING
THIS.
RDCRN FACES THE CHALLENGES,
PROGRESS IS SLOW.
TRIALS ARE EXPENSIVE, QUALITY OF
THE DATA BEING GENERATED IS NOT
HIGH ENOUGH TO USE FOR TRIAL.
WE'RE NOT JUST LOOKING AT ONE AT
A TIME, NOT JUST LOOKING AT THE
SCIENTIFIC CHALLENGE.
WE'RE NOT JUST LOOKING AT
SCIENTIFIC CHALLENGES, WE'RE
ALSO LOOKING AT ADMINISTRATIVE
AND OPERATIONAL CHALLENGES THAT
HIT THE NETWORK SO WE'RE COMING
AT PROBLEMS FROM MULTIPLE SIDES.
BY FOCUSING ON ISSUES SUCH AS
CLINICAL TRIAL READINESS,
DEVELOPED A SHARED NETWORK
ENVIRONMENT, TARGETING -- NEXT
SLIDE.
RAREDISEASESNETWORK.ORG.
BACK TO P.J.
>> THANKS, TIINA.
I'LL TRY TO GO THROUGH
HIGHLIGHTS.
ANOTHER PROGRAM YOU'RE FAMILIAR
WITH, THE INFORMATION CENTER,
ANOTHER CONGRESSIONALLY MANDATED
PROGRAM.
2.0, WE HAD A MAJOR OVERALL
BECAUSE OF SECURITY CONCERNS.
ERIC RUNS A PROGRAM WITH OTHERS.
AS WE'VE BEEN ALLUDING WE'LL BE
CONTINUING TO TRY TO FIND WAYS
TO MAKE GARD MORE OPERABLE WITH
MANY ACTIVITIES AROUND THE WORLD
WHERE THEY ARE GENERATING MUCH
OF THIS DATA.
[AUDIO DISTORTION]
GARD IS PRIMARILY INFORMATION
SOURCE WE'RE SEEING A LOT MORE
EFFORTS IN RARE DISEASE SPACE
INCLUDING WITHIN NCATS, EWY
TALKED ABOUT SOME.
WE WANT GARD TO BE A BASE FOR
THOSE ACTIVITIES SO WE ALL KNOW
WE'RE TALKING ABOUT THE SAME
DISEASES, AND THE ONE AT THE TOP
IS SPEARHEADING THE EFFORTS WITH
OTHERS TO MAKE THESE ACTIVITIES
THE GOAL AND YOU'LL HEAR A LOT
MORE ABOUT THAT IN THE FUTURE.
ANOTHER THINK THAT'S A
CONTINUING AIR, DIAGNOSTIC
ODYSSEY, IN THE RARE DISEASE
SPACE.
I WANTED TO HIGHLIGHT THE WORK
HERE.
[AUDIO DISTORTION]
WE GOT THREE MULTI-DISCIPLINARY
GENOMIC ANALYSIS AND CLINICAL
APPROACHES, RARE DISEASE
DIAGNOSTIC ODYSSEY, AND
FOLLOWING UP ON SOME
CONVERSATIONS EARLIER REALLY
WANTED TO HIGHLIGHT THE ONE DOWN
BELOW, TRYING TO WE'VE OUT TO
ALL PATIENTS INCLUDING THOSE,
MEDICALLY UNDERSERVED
POPULATIONS, THAT IS ON THE NEXT
COHORT.
SO THE BIG CHALLENGE WE HAVE,
YOU'VE SEEN THIS SLIDE, EVER
INCREASING NUMBER OF DISEASES.
THIS IS AN IDEA --
[AUDIO DISTORTION]
INCREASING ALL THE TIME BUT
NUMBER OF APPROVED THERAPIES
LAGGING FAR BEHIND, BACK TO THE
CHRIS AUSTIN QUOTE, AT THIS RATE
IT'S GOING TO TAKE 2,000 YEARS.
VERY DIFFERENT APPROACH TO
TACKLE ALL THESE DISEASES,
PARTICULARLY BECAUSE WE KNOW
MOLECULAR BASIS, WE OUGHT TO BE
ABLE TO DO SOMETHING.
I THINK WE CAN.
I THINK THE WAIT TO DO THAT IS
MANY DISEASES AT A TIME
APPROACHES, FOCUSED ON SHARED
MOLECULAR ETIOLOGY, AND
DEVELOPING THERAPEUTIC PROGRAMS,
PLATFORMS BROADLY APPLICABLE TO
MONOGENIC DISEASES.
ONE OF THE WAYS WE'RE TRYING TO
DO THIS IS TAKE A PAGE FROM THE
ONCOLOGY SPACE WITH STATE OF THE
ART TRIAL IS DRUGS THAT TARGET
MOLECULAR ABNORMALITIES IN
DIFFERENT CANSERS IS, THEY WANT
A DRUG THAT HITS ACROSS PATHWAYS
AND WE'RE TRYING TO ADOPT TO THE
SPACE.
WE'VE GOT TWO PROJECTS.
I'D LIKE TO DRAW YOUR ATTENTION
TO THE LOWER ONE THERE.
IN A SENSE CREATING A NEW
PRIMARY DISEASE BASED ON
MOLECULAR TARGET FOR A DRUG.
THAT'S A LOT MORE EFFICIENT WAY
TO DEVELOP, IF YOU START WITH A
DRUG, FIND ALL THE PATIENTS THAT
MIGHT RESPOND AND PUT THEM ON A
CLINICAL TRIAL.
ONE OF THE THINGS WE'RE DOING IN
PARTICULAR WE'VE ASKED FOR WHERE
THERE'S DIFFERENT KINDS OF
CLINICAL OUTCOME MEASURES, ONE
OF THE BIG CHALLENGES IN
APPLYING DIFFERENT DISEASES AS
OPPOSED TO CANCER.
WE ALSO HAVE AN OPEN FUNDING
OPPORTUNITY, SAME THING, SMALL
BUSINESS SPACE.
WE WERE TALKING ABOUT
INTERACTING WITH INTERNATIONAL
GROUPS, ONE IS RARE DISEASE
RESEARCH CONSORTIUM, THAT NCATS
HAS BEEN INVOLVED WITH, AND
WITHIN THIS DEVELOPED A WORKING
GROUP ON SHARED MOLECULAR
ETIOLOGIES UNDERLYING MULTIPLE
RARE DISEASES, I'VE GOT TO GIVE
DAN TAGLE CREDIT, MANY YEARS AGO
WE MOVED IT FORWARD.
THIS WORKING GROUP FROM PEOPLE
ALL OVER THE WORLD, GOT TOGETHER
AND A MANUSCRIPT IN PRESS, KIND
OF GETTING GRANDER VIEW OF HOW
THIS APPROACH -- (INAUDIBLE).
THIS INCLUDES INDIVIDUALS IN
REGULATORY AGENCIES IN THE U.S.
AND FROM NCATS.
GOING MORE INTO GENE THERAPY AND
GENE EDITING, IF YOU THINK ABOUT
KIND OF A SIMPLIFIED VERSION OF
GENE THERAPY, YOU CAN THINK
ABOUT DELIVERY VEHICLES SUCH AS
ADENOASSOCIATED VIRUSES, LIKE
DELIVERY BOXES, WE
PRE-ADDRESSED.
STRAIGHT FORWARD TO CHANGE THE
CARGO AND DEVELOP TREATMENTS FOR
DIFFERENT MONOGENIC DISEASES BY
SWAPPING OUT.
ALSO IMAGINE IF YOU COULD DO
THAT COULD MAKE THE WHOLE
PROCESS WORK EFFICIENTLY.
THAT'S THE IDEA BEHIND PLATFORM
VECTOR PROGRAM, TRY TO TEST THE
HYPOTHESIS THE PLATFORM VECTOR
APPROACH WILL INCREASE
EFFICIENCY.
SO MOVING FORWARD WITH GENE
THERAPY, VERY RARE DISEASES ALL
UNDER STUDY AT THE NIH CLINICAL
CENTER, TWO MYASTHENIA
SYNDROMES, THE IDEA WAS TO USE
THE SAME SEROTYPE FOR ALL FOUR
AND AS WE MOVED THROUGH THE
PROCESS, INTERACT WITH CSA,
WE'LL SUGGEST DIFFERENT WAYS TO
STREAMLINE THE PROCESS.
MAYBE LESS OR NOT -- MAYBE LESS
TOXICOLOGY.
WE'LL RESPOND AND THEN WE'LL
MAKE THE INFORMATION PUBLIC,
PROFIT ENTITIES WOULD NEVER DO,
THEY ARE GIVING AWAY
INTELLECTUAL PROPERTIES, BUT
THAT'S OUR GOAL TO DEMOCRATIZE,
MAKE IT PUBLICLY AVAILABLE.
HIGHLIGHTING ONE OF THE FIRST
SUCCESSES, ON THE ORPHAN PRODUCT
DESIGNATION, ON THE PROGRAMMING.
(INAUDIBLE)
INTERACT MEETING.
WE'RE IN THE PROCESS OF MAKING
THOSE DOCUMENTS PUBLIC AS WELL.
FIRST PRE-IND MEETING,
ANTICIPATED IN MID-JULY AND WE
WILL REPEAT FOR OTHER DISEASES,
TO BE MORE EFFICIENT AND
STREAMLINED.
I WANT TO HIGHLIGHT THIS IS A
TEAM EFFORT, THERAPEUTICS
DEVELOPMENTS BRANCH, OFFICES ARE
HERE.
LIZ AND MANY OTHERS, CHUCK AND
HIS TEAM.
MOVING TO THE BESPOKE GENE
THERAPY CONSORTIUM, I WON'T GO
INTO DETAIL.
IT'S A PUBLIC/PRIVATE
PARTNERSHIP THAT IS ORGANIZED BY
THE FOUNDATION FOR THE NIH IN
CASE YOU'RE NOT AWARE.
THE FOUNDATION FOR THE NIH IS
NOT PART OF THE NIH.
IT'S A STAND-ALONE 501(C)3
ENTITY TO ALLOW THEM TO WORK IN
WAYS WE OTHERWISE COULDN'T DO.
HAPPY TO WORK WITH TIM MILLER,
PETER MARKS, ALLOWS THE WHOLE
EFFORT TO COME TOGETHER.
AND PARTNERS FROM FNIH, COURTNEY
AND BRAD.
THESE ARE ALL THE PARTNERS.
THIS IS THE LARGEST OF THE FNIH
ACCELERATED MEDICINES PROGRAMS,
YOU SEE 11 INSTITUTES AND
CENTERS, MULTIPLE FOR-PROFIT
ENTITIES AND NON-PROFITS,
INCLUDING NATIONAL ORGANIZATION
OF RARE DISORDERS, WE DID WANT
PATIENTS TO PUT INTO THIS.
AND 50- 50 CONTRIBUTION OF
PUBLIC AND PRIVATE PARTNERSHIP
FUNDING.
THERE ARE TWO MAIN COMPONENTS,
ONE FOCUSED ON BASIC BIOLOGY,
PARTICULARLY VECTOR GENERATION,
THERAPEUTIC GENE EXPRESSION.
GENE THERAPIES GOING INTO
PATIENTS.
THE BIGGEST PART OF THE PROGRAM
IS THE CLINICAL PART.
TRYING TO STREAMLINE THIS PATH
TO GENE THERAPY, TARGET FOR RARE
DISEASE AND DATA AND ANIMAL
MODELS INTO THE CLINIC, AND
DOING THINGS LIKE HARMONIZE BEST
PRACTICES.
THE NOTE AT THE BOUGHT,
SOMETHING WE'RE IN THE MIDDLE
OF, WE HAVE AN OPPORTUNITY HERE,
WITH INTERACTION WITH THE FDA TO
PROPOSE CERTAIN WAYS OF
STREAMLINING INCLUDING COMING UP
WITH A MINERAL PACKAGE OF ANIMAL
TOXICOLOGY STUDIES
THEY ARE VERY EXPENSIVE AND WHAT
WE'RE DOING HERE IS WE'RE NOT
TRYING TO CUT CORNERS AND PUT AT
RISK PATIENT SAFETY BUT WE'RE
TAKING A SCIENCE-BASED APPROACH
TO ASK WHAT ARE TOXICOLOGY
STUDIES TELLING US AND WHAT DO
WE NEED TO KNOW TO GET THESE
TRIALS UP AND RUNNING QUICKLY
AND SAFELY.
WE'LL HAVE A CONVERSATION WITH
FDA AS MOVE FORWARD.
WE NEED TO DO BETTER GENE
THERAPY TRIALS, LIKE THE AGT.
AND WE HAD AN OPEN NOMINATION
PROCESS, WE GOT 62 APPLICATIONS.
NEARLY ALL WOULD HAVE BEEN A
GOOD FIT.
SO INDICATION OF THE GREAT NEED
IN THIS SPACE.
THERE WAS INITIALLY CUT OUT FOR
14, WE SELECTED 8 TO GO FORWARD,
FIRST IN HUMAN TRIALS.
AND THIS IS THE SEVEN DISEASES
WE GIST ANNOUNCED LAST WEEK
ACTUALLY.
THREE OCULAR, THREE NEUROLOGIC.
WE'RE AT THE POINT OF SETTING UP
AND STARTING UP THE EIGHT
TRIALS, BOTH EXCITING AND
SOMETHING TO THINK ABOUT.
LOOKING FORWARD TO THIS
CHALLENGE.
FINALLY I'LL MOVE TO THE SOMATIC
CELL GENOME EDITING PROBLEM
THAT'S FUNDED BY THE COMMON
FUND.
IT WAS FOCUSED ON DEVELOPING
NOVEL TECHNOLOGIES AND BUILDING
THE FOUNDATION FOR CLINICAL
TRIALS, GENOME EDITING GOING
INTO THE CLINIC.
PRODUCTER SON COURT --
CONSORTIUM.
A BIG CHUNK FROM THE WORK BY
DAVID LU, THE PRIME EDITOR.
THE COMPANY HAS SPUN OUT OF
WORK, AN THAT'S PHASE 1.
WE ALSO FUND THE TOOL KIT AT
UNIVERSITY OF WISCONSIN, LINKS
SHOWN HERE.
YOU CAN GO INTO THE TOOL KIT AND
LOOK FOR THE MODEL SYSTEMS,
DELIVERY SYSTEMS, ET CETERA.
PART OF THE PROGRAM WE FUNDED 20
BOARDS ON WAYS TO DELIVER GENOME
EDITING.
WE'VE ENCOURAGED PEOPLE TO DO
EVEN EXPERIMENTS THAT DIDN'T
WORK.
I WANT TO ENCOURAGE PEOPLE TO
PUT THIS DATA INTO THE PUBLIC
DOMAIN IN A WAY.
SOMETHING ELSE NOVEL WE PUT A
REQUIREMENT FOR INDEPENDENT
VALIDATION OF THE NEW DELIVERY
TECHNOLOGY.
THESE ARE THE TWO PHASE AWARDS
AND THE KEY POINT WAS TO GO FROM
THE FIRST FACE OF THE WORK IN
THE INVESTIGATOR'S LAB TO
SCALEUP PHASE REQUIRING
INDEPENDENT VALIDATION AND
FUNDING TO DO THAT.
HERE'S ONE EXAMPLE HOW THAT
WORKED.
AT THE TOP WORKED WITH THE
INVESTIGATOR'S LAB, AND THOSE
ARE THE CELLS, ON THE RIGHT WITH
TOMATO, 20% OF CELLS ARE EDITED
IN THE INVESTIGATOR'S LAB.
THAT WAS MADE BY THE SAME SYSTEM
BAYLOR COLLEGE OF MEDICINE
ACKNOWLEDGING QUITE CLOSE.
THIS WORK WAS PUBLISHED.
YOU CAN SEE THE PUBLICATION
INCLUDES ORIGINAL INVESTIGATORS
AS WELL AS THE FOLKS FROM THE
VALIDATION CENTER AND TWO CLOSED
SETS OF DATA, WHICH I THINK IS A
POWERFUL WAY TO --
NOW MOVING TO PHASE 2, AND
MOVING TOWARDS THE CLINIC.
WE HAVE SEVERAL INITIATIVES
HERE, ONE ON TRYING TO DEVELOP
ASSAYS WITH A LEVEL OF RIGOR AND
REPRODUCIBILITY, BECOMING PART
OF THE REGULATORY PROCESS.
YOU CAN'T CONTROL THAT OBVIOUSLY
BUT WE HAVE WORKED WITH THE FDA
TO GET UNDERSTANDING FROM THEM.
WE'LL HAVE A LARGE GROUP TO
STUDY FOCUSED ON GETTING TO AN
IND FOR GENOME EDITING,
DIFFERENT DISEASE STATES WITH
THE IDEA THEY COULD BE PICKED UP
BY INDUSTRY, SIMILAR TO THE
TOOLKIT FROM PHASE 1.
ONE OTHER INITIATIVE, IT'S
FOCUSED ON SUPPORTING PLATFORM
CLINICAL TRIALS OF GENOME OH DID
ITERS, PUP DISEASES.
IT'S A THERAPEUTIC PRATT FORM,
BUT A TYPICAL WAY, YOU WANT TO
ENCOURAGE THE IDEA OF DEVELOPING
THERAPEUTIC PLATFORMS AS
THERAPEUTIC PLATFORMS FROM THE
BEGINNING, AND THAT'S WHY WE PUT
OUT THAT TERMINOLOGY PAPER.
AND I HOPE TO MAYBE TELL YOU
MORE AT THE NEXT COUNCIL ABOUT
HOW THAT WORKED OUT.
AND FINALLY ONE THING WE'RE
EXCITED ABOUT IN PHASE 2 IS TO
DO A LITTLE BIT MORE OR NEW
APPROACH TO DEVELOPING.
PHASE 1 WE FUNDED ONE, HERE THE
IDEA TO DO A PRIZE CHALLENGE.
WHICH WE CAN DO AT NIH, FOCUSED
ON TWO AREAS.
ONE WOULD BE PROGRAMMABLE
DELIVERY SYSTEMS, WHERE YOU
DEVELOP PROGRAM TO BUILD
DISTINCT CELL TYPE TISSUE AND
ORGANS.
AND THE OTHER IS LOOKING FOR
HIGHLY EFFICIENT NON-VIRAL
DELIVERY SYSTEMS ACROSS THE
BLOOD-BRAIN BARRIER.
MAJORITY OF TARGET CELL TYPES,
AND THIS ONE, THE WAY YOU SET IT
UP TOP COMPETITORS GO UP TO A
MILLION DOLLARS IN PRIZE MONEY.
AS WITH PHASE 1 WILL INCLUDE
VALIDATION.
GOT AN INFORMATIONAL WEBINAR
NEXT WEEK.
THERE'S THE CODE.
BY ALL MEANS, LET FOLKS KNOW.
WE'RE HOPING IN ADDITION TO
TRADITIONAL ACADEMICS TO GET
MORE DIVERSE SET OF APPLICANTS,
SO THERE'S THE PRIZE CHALLENGE.
FINALLY TO WRAP UP, THERE'S A
BIT OF A SQUARE PEG IN A ROUND
HOLE.
EVERYTHING WE DO, THE BIOMEDICAL
SPACE FROM DEVELOPINGMENT TO
REGULATORY RY PATH, YOU LEARN
SCREENING.
ALMOST ALL IS DEVELOPING
TREATMENT FOR INDIVIDUAL
DISEASES.
NOW WE HAVE THERAPEUTIC
PLATFORMS THAT ARE APPLICABLE IN
PRINCIPLE TO MANY DIFFERENT
MONOGENIC DISEASES.
AND THOSE TWO CONCEPTS REALLY
DON'T COME TOGETHER VERY WELL.
BUT I THINK IF WE EVER WANT TO
GET TREATMENT TO ALL OF THE
PATIENTS THAT CAN BENEFIT FROM
THEM, WE HAVE TO DEVELOP
THERAPEUTIC PLANS.
ABOUT A YEAR AND A HALF AGO NOW,
WE HAD A LARGE MEETING AT NIH.
ACTUALLY A SERIES OF MEETINGS,
THESE ARE VIRTUAL OF COURSE IN
2021.
LED BY TIINA URV, BROUGHT
TOGETHER PEOPLE DEVELOPING
TECHNOLOGIES TO MAKE PEOPLE
AWARE OF WHERE THIS FIELD IS
GOING.
A LOT OF THIS GOES BEYOND WHAT
WE CAN DO AT NIH BUT WE WANTED
TO CONTINUE.
WE HAVE PEOPLE TO THINK ABOUT
IT, MAYBE GOD AROUND TO
PUBLISHING WITH LEADERS FROM ALL
THESE ACTIVITIES WHICH IS
INTERESTING.
I WANT TO THANK TIINA, JOANNE,
MELISSA FROM NICHD FOR ALL THAT
WORK.
AND HIGHLIGHT ALL THE PEOPLE,
THE WONDERFUL COLLEAGUES AT
DRDRI, SO MANY IT'S HARD TO
MENTION.
AND I'M HAPPY TO TAKE ANY
QUESTIONS.
>> THANK YOU, P.J.
ANY QUESTIONS FOR P.J.?
OR TIINA?
>> I DON'T HAVE A QUESTION,
JUST THANK YOU FOR ALL THE WORK
YOU'RE DOING.
RDCRN CONTRIBUTED TO EIGHT
TREATMENTS FOR RARE DISEASES, A
SLIDE IN YOUR PROGRAM.
I FEEL LIKE THAT NEEDS TO BE
SPLASHED ON BUSES, BILLBOARDS,
I'M NOT JOKING.
THAT'S PHENOMENAL.
JUST THE YIELD, UNDERSCORING THE
POWER OF THE WORK.
THANK YOU FOR ALL THAT WORK.
>> THANK YOU FOR THAT COMMENT.
I COMPLETELY AGREE AND WE'RE
TRYING TO FIGURE OUT HOW CAN WE
DO MORE OF THIS WITHIN OUR
DCCRN, YOU KNOW, WE'RE LIMITED
BY OUR RESOURCES BUT I THINK
THAT THE PROGRAM IS BECOMING
MORE MATURE AND LESSONS WE'VE
LEARNED ALONG THE WAY HAVE BEEN
VALUABLE.
SO THAT'S THE HOPE TO PUSH THAT
IDEA.
MATTIAS, GO AHEAD.
>> ALSO WHAT WE'VE SEEN IN THE
PRESENTATION AS WELL, LESSONS
LEARNED IN THE FIELD BY RDCRN,
FORMALIZED AND SHARED WIDELY.
P.J.
I WANT TO ENCOURAGE YOU AS YOU
HAVE TOOL SETS OUT THERE WHERE
YOU TAKE THOSE AND MAKE THEM
ACCESSIBLE TO THE COMMUNITY AT
LARGE, THAT'S AN EFFORT YOU
CONTINUE TO WRITE IN OUR TOOLBOX
AND MAKE SURE YOU HAVE THE
SUPPORT AND IP INFRASTRUCTURE IN
PLACE TO DO THAT.
BECAUSE THEN OBVIOUSLY THE
SOLUTION WORKING FOR A SMALL SET
OF RARE DISEASES, IN A WIDE
SPECTRUM OF DISEASES AS WELL,
AND OUR PATIENT INTEREST CLOSELY
ALIGNED, OBVIOUSLY A KEY
DISTRIBUTION, DOING QUITE WELL
AND HOPEFULLY WITH YOUR TEAM AND
FACILITATE THESE EFFORTS GOING
FORWARD.
>> THANKS, MATTIAS.
>> I HAVE A QUESTION.
SO, THE OVERVIEW DISRUPTION THAT
YOU'RE TALKING ABOUT, AND I FEEL
LIKE NOBODY IS THINKING OR
REACTING SO IT'S CONTROVERSIAL
TO IMAGINE A WORLD WHERE COMMON
PATHWAY CAN BE AN APPROACH TO
REGULATORY APPROVAL.
WHAT NEEDS TO CHANGE IN THE REAL
WORLD WITH FDA TO MAKE THAT
HAPPEN?
I FEEL LIKE YOU ALLUDED, LIKE
YOU KNOW MORE AND I WOULD LOVE
TO UNDERSTAND WHAT REALLY NEEDS
TO HAPPEN SO WE COULD ALL APPLY
IN OUR PATIENT COMMUNITIES.
>>A COUPLE THINGS.
IN TERMS OF SHARED MOLECULAR
PHYSIOLOGY, I'M NOT SURE THE
REGULATORY -- THERE IS SO MUCH
REGULATORY CHALLENGE.
THESE ARE COOPERATIVE
AGREEMENTS, WHICH MEANS THAT I
GET TO BE INVOLVED.
(INAUDIBLE)
CONVERSATIONS I HAD, THAT'S MY
IMPRESSION.
IN TERMS OF BIOLOGICS, THAT'S A
WHOLE DIFFERENT DEAL.
>> RIGHT.
>> THAT'S JUST NOT THE WAY
PEOPLE ARE THINKING ABOUT THESE
THINGS.
AND I THINK IN PART, THE IMPETUS
ISN'T THERE, IT'S NOT THE WAY
PEOPLE THINK, EVEN THE WAY THEY
THINK ABOUT CALCULATING, SO I
THINK THAT'S PARTLY WHY WE FEET
WE NEED TO FUND THESE THINGS.
SO THEY FUND PEOPLE, THEY GO
FORWARD, HOW WELL THAT WORKS, SO
IT'S REALLY TO FUND SOME OF
THESE THINGS, INTERACT WITH THE
FDA AND COMMUNICATE BACK.
>> IF I COULD OFFER COMMENTARY,
IF YOU'RE TALKING ABOUT ONE DRUG
THAT THESE MANY, JUST AFFECTING
MULTIPLE APPLICATIONS THE FDA IS
COMFORTABLE WITH MOVING DRUGS
FORWARD IN THAT REGARD.
I DON'T THINK THERE'S ANY MAJOR
ISSUES OTHER THAN THE FDA MAY
HAVE A CONCERN THERE MY BE
UNIQUE ADVERSE EVENTS, IN ONE
INDICATION VERSUS NOT THE OTHER,
THAT'S THE ONLY THING YOU WOULD
BE CONCERNED WITH BESIDES HAVING
DEFINITE EFFICACY, PJ WAS
TALKING ABOUT FROM A
MANUFACTURING PLATFORM IT'S NOT
THAT FDA HAS AN ISSUE WITH IT.
IT'S JUST THEY HAVE DONE THIS,
TO A CERTAIN EXTENT THE FDA WITH
PERFECTLY COMFORTABLE.
HOW IS IT WE COME UP WITH A
BRAND-NEW FLU VACCINE EVERY
YEAR?
IT'S A UNIQUE PRODUCT CHANGED
EVERY YEAR BUT THE FDA FULLY
ACCEPTED THE MANUFACTURING
PROCESS FOR GENERATING THAT.
SO AS LONG AS YOU DEMONSTRATE
THAT YOU HAVE THE CORRECT ASSAYS
AT THE END, CHECK TO SEE THAT
WHAT YOU GOT OUT IS WHAT YOU
THOUGHT YOU WERE PUTTING THROUGH
THE PROCESS, THE FDA IS
PERFECTLY COMFORTABLE MOVING
FORWARD.
THAT'S THE IDEA IS DEMONSTRATE
THE MANUFACTURING PLATFORMS OF
THESE COMES OUT WITH THE SAME
RESULT OVER AND OVER AGAIN.
YOU'RE STILL GOING TO HAVE TO
DEMONSTRATE THE INDIVIDUAL
PRODUCT WORK IN THE PARTICULAR
DISEASE.
>> YOU START TALKING ABOUT
TREATING MANY DISEASES, WE
TALKED ABOUT A SCIENCE-BASED
APPROACH.
WE NEED A DIFFERENT REGULATORY
PATH.
WE HAVE DISCUSSIONS.
>> PART OF THE -- (INAUDIBLE)
IT'S JUST THERE'S MORE THAN JUST
THE FDA.
THERE'S A LOT OF DIFFERENT
COMPONENTS TO BRING THE PEOPLE
AROUND THE TABLE.
>>I THINK IT SEEMS ONE COULD
MAKE -- (INAUDIBLE).
>> TOPIC FOR FURTHER
DISCUSSION.
THE EXAMPLE YOU USED, THE JACK
PATHWAY EVERYTHING IS 1:1, IT'S
NOT ONE TO MANY.
ONE TO MANY THAT YOU ARE
PROPOSING IS JUST SOMETHING --
WE HAVE TALENTED
RHEUMATOLOGISTS, THE BEST
PHARMA.
PHARMA DOESN'T HAVE THE
INCENTIVE.
IT'S A NEW KIND OF -- AS A
PATIENT, SOMEBODY WHO REPRESENTS
MANY PATIENTS, I -- I DON'T
THINK THAT -- MAYBE IT'S AN
AWARENESS ISSUE THAT FDA WILL --
IS AMENABLE TO --
>>THE EXAMPLES PJ HIGHLIGHTED
IS RELEVANT, THE FDA HAS DONE
THIS FOR AN ONCOLOGIC
INTERVENTION WHERE YOU DID NOT
COME IN WITH LIVER CANCER OR
LUNG CANCER OR SOMETHING.
YOU CAME WITH WITH ANY CANCER
WITH THIS MOLECULAR ETIOLOGY,
AND THE DRUG WAS DESIGNED TO
AFFECT THAT.
SO ALL COMEERS CAME IN AND THEN
THE FDA PASS APPROVED THAT
PRODUCT, SO THEY WORKED THROUGH
THE MECHANISM FOR DOING IT IN
ONCOLOGY, WHICH SORT OF ALWAYS
GETS A -- (INAUDIBLE).
>> WE CHALLENGED THEM TO SAY
LET'S SEE YOU DO IT MANY WAYS
AND MAKE THAT IT.
>> THE ONCOLOGY OUTCOME, ARE
THEY LIVING OR DEAD?
IF YOU'RE LOOKING AT RARE
DISEASES, UROLOGICAL DISORDERS,
IT'S NOT SO EASY TO TELL
SOMEBODY, IT'S CHALLENGING.
>> IT IS A SHIFT IN THINKING
BECAUSE I CAN TELL YOU AS A
PATHOLOGIST, IN EVERY TUMOR IF
YOU FOUND METASTASIS, IT'S
ALWAYS WHAT'S THE PRIMARY?
THAT'S THE BASIS FOR TREATMENT.
IT'S NOT WHAT IS THE PRIMARY NOW
BUT WHAT IS THE MOLECULAR
SIGNATURE OF THE TUMOR TYPE.
IT IS AN EVERY LUGES IN --
EVOLUTION IN THE ZEALS PROCESS.
>> MANY OF THE ELEMENTS WE'VE
SEEN, IN YOUR STRATEGIC
STATEMENT, THIS IS THE ADVANTAGE
OF NCATS.
PLEASE TO CONTINUE TO PUSH THAT.
YES, YOU HAVE PLATFORMS IN PLACE
YOU USING TOOLS AND BLOCK FORMS.
YOU HAVE THE I.P. IN PLACE, TO
BRING THOSE BACK, AND WITH
REVERSE YOU REQUEST DO REVERSE
AND IDENTIFY PHENOTYPES.
JAK/STAT WAY, THEY, EVER ARE ARE
ARE ARE ARE ARE -- PATHWAY,
WE'RE TREATING PEOPLE WITH THE
EXCESSIVE RISK FOR KIDNEY
DISEASE USING MOLECULAR
FINGERPRINTING APPROACH FROM
COMMON DISEASE TO RARE DISEASE
IN ACQUIRED STATE.
IT'S A MINDSET WHICH GOES INTO
RARE DISEASE FIELD WHERE YOU
MADE SIGNIFICANT PROGRESS
TOWARDS COMMON DISEASE, RISKY
WAY TO LEAVE BEHIND DISEASES IN
MECHANISTIC TERMS, STARTING WITH
GENES BUT UNDERSTANDING THAT
GENETIC APPROACHES ARE NOT THE
ONLY OPPORTUNITY TO DO THAT BUT
MORE INTERMEDIARIES AS YOU
NICELY ALLUDED TO EARLIER THIS
AFTERNOON, KEY ELEMENT TO MAP
WELL TO CLOSE THE GAPS.
WE FOUND IT.
REGULATORY AGENCIES, THE
OUTCOMES OBVIOUSLY HAVE TO STAND
UP AND THOSE HAVE TO BE THE NO
AND MONEY SPECIFIC BUT FROM THE
OTHER APPROACHES -- (INAUDIBLE).
>>I'LL ADD -- THANKS FOR THAT,
MATTIAS.
I THINK THAT'S RIGHT.
THE OTHER PIECE IS THAT OUTCOMES
OF COURSE IS THE BIGGEST THING
BUT THE OTHER PIECE IS
DEVELOPING THE REGULATORY PATH
FOR THIS IDEA OF PRECEDENT
SETTING AND HOW TO MAKE SURE
THESE DELIVERY MECHANISMS WILL
BE WORKABLE IN DIFFERENT
SCENARIOS.
SO WHAT I THINK PJ'S PROGRAMS
ARE WORKING TOWARDS IS PROVIDING
THAT PRECEDENT-SETTING PROCESS
THAT WILL ENABLE THE REGULATORY
PATH BECOME MORE CLEAR FOR FDA
WORK AND BEYOND.
>>TOTALLY.
>> DEFINITELY WORKING TOWARD
THAT SPACE.
DID I SEE ONE OTHER HAND UP AS
WELL?
I MAY HAVE MADE IT COMPLETELY UP
BUT I THOUGHT I SAW ANOTHER
HAND.
OKAY, MAYBE NOT.
ANY OTHER COMMENTS?
>> I SEE SOME LOOKING AT TARGET
AND CHALLENGE.
>> EXCELLENT.
YES.
KEITH, DID YOU HAVE A COMMENT?
>> NO.
>> OKAY.
GREAT.
WELL, P.J. AND TIINA, THANK YOU
SO MUCH.
VERY MUCH FOR ALL OF THAT WORK.
IT'S FUNNY, WHEN YOU MAKE A LOT
OF NOISE IN THE ROOM IT GOES
SILENT ON OUR END.
I KNOW YOU WERE CLAPPING.
WE HAVE A LITTLE BIT MORE TIME.
I'M RECOGNIZING TOO THAT YOU
STARTED VERY EARLY TODAY.
I KNOW THAT BECAUSE OF THE TOUR.
SO I WANT TO GAUGE YOUR SORT OF
ENTHUSIASM FOR PRESSING ON.
RECOGNIZING IT IS NEARING THE
END OF THE TIME HERE.
I'M GOING TO ASK YOU, NUMBER
ONE, ARE YOU OKAY WITH A FEW
MORE MINUTES?
OR WE CAN ALSO CONTINUE SOME OF
THESE CONVERSATIONS CERTAINLY
NEXT TIME.
BUT IF I CAN HOLD YOU FOR
PERHAPS FIVE MORE MINUTES OR SO,
I'D LOVE TO CIRCLE BACK TO THIS
IDEA OF THE STRATEGIC PLANNING
QUESTIONS AND ENGAGING YOU JUST
A LITTLE BIT MORE, REALLY MORE
PICKING YOUR BRAIN AND YOUR
EXPERTISE AND EXPERIENCE AND
LEADERSHIP POSITIONS THAT YOU
HOLD.
WHAT IT MEANS TO YOU TO SORT OF
PUT TOGETHER A STRATEGIC PLAN,
PROGRAM, AND RECOGNIZING THE
INFORMATION THAT WE PRESENTED TO
YOU TODAY FROM MEREDITH AND
PENNY AND ALL OF THE INPUT THAT
WE RECEIVED, AS WE'RE STARTING
TO SORT OF BUILD THAT TOGETHER
AND PUT IT ALL TOGETHER, THE
ISSUE AROUND PRIORITIZATION TO
ME IS A BIG ONE.
AND SO I WOULD LOVE TO HEAR FROM
YOUR EXPERTISE AND YOUR
EXPERIENCE THINKING ABOUT LARGE
PROGRAMS AND AREAS TO MOVE
FORWARD.
THE WHAT AND HOW.
WHAT TO YOU IS MOST IMPORTANT
FOR THAT PRIORITIZATION FOR US,
GIVEN ALL THE VARIED INPUT THAT
WE HAVE AND VAST TRANSLATIONAL
SCIENCE SPECTRUM THAT WE
SUPPORT?
AND SECONDLY, SORT OF HOW DO WE
NEED TO BE THINKING ABOUT
PRIORITIZATION PROCESS.
AND I'LL JUST GIVE A SENSE OF
WHAT I'M THINKING ABOUT.
IN TERMS OF THAT PRIORITIZATION,
NUMBER ONE, I THINK -- THERE ARE
SEVEN POINTS.
NUMBER ONE, I'M THINKING THAT
SORT OF HAVING THE RELEVANCE TO
THE SORT OF STRATEGIC GOALS AND
MISSION OF NCATS IS CERTAINLY
FIRST AND FOREMOST.
AND HOW WE CAN ALIGN THE
DIFFERENT KINDS OF INPUT TO THE
MISSION AND VISION, THAT SEEMS
TO BE ONE AREA.
THE SECOND THING TO BE THINKING
ABOUT THE IMPACT, WHAT'S GOING
TO BE THE POTENTIAL IMPACT AND
INFLUENCE THAT WE CAN PROVIDE
BASED ON THE INFLUENCE THAT WE
RECEIVED AND PUT THAT INTO THE
FRAMEWORK.
AND THAT WILL HELP US CONTRIBUTE
TO OUR LONGER-TERM VISIONS.
THOSE MAY NOT NECESSARILY BE
SHORT TERM BUT HOW DO WE MAKE
SURE WE INFUSE THEM TO CONTINUE
THE LONG GAIN OF DEVELOPING THAT
VISION AHEAD?
AND THE THIRD PIECE IS THINKING
ABOUT OF COURSE FEASIBILITY AND
RESOURCES.
OBVIOUSLY WE WORK IN A ZERO SUM
GAIN SCENARIO AWAY.
VERY ANNUAL APPROPRIATIONS, ALL
OF THOSE TYPES OF THINGS.
BUT IT'S THOSE RESOURCES THAT
ARE FINANCE, THEY ARE ALSO HUMAN
RESOURCES AND TECHNOLOGICAL
RESOURCES, HOW DO WE MAKE SURE
THOSE ARE ALIGNED ALSO WITH
BEING ABLE TO SUPPORT THE
VISION.
SO THAT SEEMS TO BE ANOTHER AREA
THAT WE'LL NEED TO USE IN OUR
PRIORITIZATION, THINKING ABOUT
RISKS, FOR EXAMPLE, WHAT ARE
THINGS THAT MIGHT BE MORE RISKY
FOR US?
SOME
SOME OF US LIKE TO TAKE A RISK,
NOT A BAD THING, HOW DO WE NEED
TO PRIORITIZE?
WE ALSO -- I THINK SOMEONE MADE
THE COMMENT EARLIER, THINKING
HOW TO REACH BACK TO
STAKEHOLDERS AND ENGAGE THEM AS
WE CONTINUE TO MOVE FORWARD,
THIS IDEA OF THE SMALL
COMMUNICATIONS ALONG THE WAY
MIGHT BE ONE OF THE WAYS TO DO
THAT.
REALLY UNDERSTANDING HOW IT CAN
CONTINUE TO GET INPUT ALONG THE
WAY WILL BE IMPORTANT AS WELL.
I GUESS ALONG THOSE SAME LINES,
GETTING THAT INPUT, IS THE
ABILITY TO MEASURE OUR PROGRESS,
IF YOU WILL, IN OUR STRATEGIC
PLANNING PROCESS.
SO THAT MEASUREMENT AND
MONITORING OF OUR ACTIVITIES AND
HOW WE CAN COMMUNICATE THAT WELL
I THINK IS ALSO AN AREA THAT WE
WANT TO HIGHLIGHT.
I SAID SEVEN, I'M NOT SURE I
WENT TO SEVEN.
I MAY HAVE MISSED SOMETHING.
THOSE ARE THE KINDS OF KEY
POINTS THAT I WANTED TO THROW
OUT FOR MAYBE JUST ROUNDING OUT
THE DISCUSSION OF THE DAY AND
TO -- WHILE I HAVE YOU, CONTINUE
TO GET YOUR SAGE ADVICE MOVING
FORWARD AS WE START TO GO INTO
THE NEXT PHASE OF OUR STRATEGIC
PLAN PROCESS.
MEREDITH PENNY, ANYTHING TO ADD
BEFORE WE MOVE ON AND ASK FOR
COMMENTS?
>>WELL, I THINK WE COULD PUT A
MAILBOX INTO THE CHAT.
IF YOU WANT TO COGITATE AND SEND
IT TO US WE WILL TAKE IT.
THE OTHER THING I WANTED TO
EMPHASIZE, I THINK I SAID THIS
ONCE DURING THE PRESENTATION,
WE'RE NOT DONE YET GETTING YOUR
INPUT.
WE'LL BE PUTTING OUT THIS SUMMER
A REQUEST FOR INFORMATION SO
PEOPLE CAN PROVIDE AS MUCH
DETAIL AS THEY WANT ON THEIR
COMMENTS.
WE HAVEN'T DRAFTED WHAT THAT RFI
LOOKS LIKE YET.
MY EXPECTATION IS WE WILL TRY TO
SUMMARIZE WHAT WE'VE HEARD TO
DATE BECAUSE I DON'T THINK WE
NEED TO GO BROADER.
EVERYTHING IS WE NEED TO GO
DEEPER TO REALLY UNDERSTAND AND
GET AT WHAT ARE THE SCIENTIFIC
AND OPERATIONAL ISSUES THAT ARE
HOLDING US BACK.
OR TO GET MORE INFORMATION ON
THE OPPORTUNITIES THAT WE REALLY
NEED TO PAY ATTENTION TO.
WE'VE GOT A TON OF FEEDBACK.
IT'S BEEN INFORMATIVE AND I
DON'T KNOW IF I'M SURPRISED BUT
IT'S REALLY INTERESTING HOW AT A
CERTAIN LEVEL IT'S VERY
CONSISTENT, WHAT WE'RE HEARING
ABOUT THINGS WE NEED PAY
ATTENTION TO.
WE'RE NOT DONE YET.
AS WE DID AT THE ROUNDTABLE,
WE'RE TRYING TO GIVE YOU AS MANY
OPPORTUNITIES TO TELL US WHAT
YOU THINK WE NEED TO FOCUS ON
THROUGH OUR RFI, WE HAVE A
MAILBOX, I HEARD A LOT OF
SUGGESTIONS FOR ADDITIONAL
ROUNDTABLES, BUT AT SOME POINT
WE'LL HAVE TO PUT PEN TO PAPER
AND GET THIS DONE.
MEREDITH, DO YOU HAVE ANYTHING?
>>NO, I THINK YOU HIT THE NAIL
ON THE HEAD, PENNY.
WE'RE TRYING TO THINK HOW DO WE
KEEP HONING THIS AND GOING
DEEPER.
ONE OF THE THINGS WE'RE GOING TO
BE THINKING ABOUT IS WHAT LEVEL
WITHIN THE STRATEGIC PLAN IN
TERMS OF DEPTH AND SPECIFICITY
DO WE WANT TO STRIKE.
SO WE'LL BE THINKING ABOUT THAT
AS WELL AND WELCOME YOUR
THOUGHTS FROM THAT PERSPECTIVE
AS WELL.
>> ROBIN, I SEE YOUR HAND.
PLEASE GO AHEAD.
>> YES, THANKS.
I'VE BEEN VERY IMPRESSED SO FAR.
PERHAPS AS YOU MOVE FORWARD,
OPERATIONALLY, YOU WANT TO --
NCATS IS DOING THING FASTER, THE
MANTRA.
IF YOU OPERATIONALIZE THE PARTS
OF THE STRATEGIC PLAN, IF THERE
ARE WAYS YOU COULD STREAMLINE
PROCESSES FOR HOW PEOPLE
INTERACT OR THE FACT THAT IT
TAKES 18 MONTHS TO GET SOMETHING
DONE.
IN OUR CURRENT WORLD AND WITH
COVID WE NEEDED SUCH RAPID
RESPONSES.
AND ARE THERE WAYS THAT -- AND I
KNOW WE MAY THIS MAY NOT BE
WITHIN YOUR CONTROL BUT
FACILITATING PROCESSES TO ALLOW
FOR TRULY TIME SENSITIVE RAPID
RESPONDING THAT DOESN'T TAKE TWO
YEARS TO GO FROM IDEA TO
IMPLEMENTATION AND BY THE TIME
IT'S DONE, IT'S IRRELEVANT.
>> IT'S A GREAT POINT.
IT USED TO BE A COUPLE YEARS AGO
THAT NCATS WAS THE ONLY I.C.
THAT USED THE SORT OF NEWER
MECHANISM CALLED THE OTHER
TRANSACTION AUTHORITY WHICH WAS
SUPPOSED TO HELP THINGS MOVE
FASTER, AND WE HAD THAT
AUTHORITY THROUGH THE CURES
ACCELERATION NETWORK PROGRAM BUT
IT WAS CAPPED AND STILL IS
CAPPED.
SO WE CAN ONLY USE IT TO A
CERTAIN DEGREE.
WE'RE HOPING -- THOSE ARE THE
KINDS OF THINGS WE'RE LEARNING
FROM.
IF THERE ARE WAYS WE CAN START
TO FIGURE OUT HOW TO EXPAND
THAT, THAT'S ONE SMALL EXAMPLE.
THERE ARE MANY OTHERS WHERE I
THINK WE HAVE LESSONS LEARNED WE
NEED TO MAKE SURE WE CAN APPLY.
SO, YEAH, I TAKE YOUR POINT VERY
WELL THAT NOT LOSING SITE ON
HELPING WITH THE PROCESS PIECE
IS IMPORTANT.
ANNA, DID YOU HAVE A COMMENT ON
THAT?
>> AS DIRECTOR OF DIVISION OF
EXTRAMURAL ACTIVITY I CAN SAY
WITH CONFIDENCE WE CAN IN FACT
MOVE QUICKLY.
AND WE CAN STREAMLINE THE
PROCESS.
ALWAYS HAVE THOSE OPTIONS.
I THINK UNDER COVID WITH
FLEXIBILITIES WE MAXED OUT.
THE ISSUE IS WHETHER WE CAN
CONTINUE TO MOVE AS EXPEDITIOUSY
WITHOUT THAT.
THE ANSWER IS MORE THAN WE WOULD
THINK.
THE ISSUE IS WHETHER OR NOT WE
HAVE THE SURGE CAPACITY AND
WHETHER IT'S SUSTAINABLE.
>> WE ALL EXPERIENCED THAT AT
UNIVERSITIES, WE HAD TO DO
EXACTLY THAT.
THERE ARE A LOT OF LESSONS WE
LEARNED.
MAYBE I DIDN'T NEED TO DO THIS.
WE'VE LEARNED FROM THAT.
>>ONE THING I WOULD SAY THAT I
DIDN'T HAVE A CHANCE TO SAY
BEFORE I WAS LOOKING AT THE
STAKEHOLDERS THAT HAVE INTN
ENGAGED TO CONTRIBUTE TO THE
STRATEGIC PLAN, IT DIDN'T SEEM
TO ME THAT THE LEADING VENTURE
CAPITAL FIRMS WERE SPOKEN TO AND
I THINK THAT THEY ARE AN
IMPORTANT VOICE TO CONSIDER
BECAUSE NCATS HAS SUCH AN
INCREDIBLE REPUTATION FOR
ADVANCED TECHNOLOGIES, THE TOP
TIER B.C.s ARE MAKING OVER THE
HORIZON TECHNOLOGY BETS THAT
UNDERSTANDING WHAT THEY ARE
SEEING, IT WOULD BE INFORMATIVE.
>> I'M ASSUMING THAT'S KELLY.
I CAN'T SEE YOU, BUT I HEAR YOU.
I WOULD LOVE TO FOLLOW UP WITH
YOU ON THAT.
>> OKAY.
>>SO STAY TUNED.
>>OKAY.
SOUND GOOD.
>> THANK YOU.
PENNY?
>> WE CERTAINLY NEED TO -- WE
COULD CERTAINLY USE SOME HELP ON
DIFFERENT STRATEGIES FOR
OUTREACH AS WE CONTINUE ON TO
MAKE SURE THAT WE DON'T LEAVE
ANYBODY BEHIND.
>> HAPPY TO HELP IN THE AREAS
WHERE I'M ABLE TO.
>> AND ANNIE?
>> SO, I WILL CERTAINLY RESPOND
TO THE MAILBOX, PARTICIPATE IN
FUTURE CONVERSATIONS BUT MAYBE A
COUPLE THINGS TOP OF MIND RIGHT
NOW, TO YOUR LIST OF QUESTIONS,
JONI.
MY FIRST RESPONSE TO THE
QUESTION AROUND RISK IS TAKE
RISKS.
FAIL FAST.
THIS IS THE PLACE THAT CAN
HAPPEN.
AND THIS IS THE PLACE WHERE WE
DE-RISK THE ENVIRONMENT,
STAKEHOLDERS OUTSIDE OF HERE.
THAT WOULD BE MY GUT RESPONSE TO
THAT.
AND THEN I WANT TO -- I JOTTED
DOWN, JUST SOME QUICK NOTES FROM
THINGS WE'VE HEARD TODAY,
HIGHLIGHTS FROM THINGS SEEN AND
HEARD TODAY RELATING TO EXACT.
NOT COMPREHENSIVE, JUST FROM MY
LENS.
DCRN CRATED TO EIGHT TREATMENTS,
CURE I.D. IS LEADING IN
PARTNERSHIP WITH FDA.
WE GET TO DO THE SCIENCE, PHARMA
ISN'T ALLOWED TO DO.
WE'VE BEEN EDING ANTI-VIRAL
PROGRAMS IN PREPAREDNESS FOR
FUTURE PANDEMICS.
SAW IT DOWNSTAIRS, A NUMBER
OF -- I'M GOING TO CALL THEM
AMAZING ROBOTS, INCREASING
ACCURACY AND EFFICIENCY, SAVING
THE ENVIRONMENT, REDUCING WASTE,
CONTRIBUTING TO CLINICAL TRIALS
THROUGHOUT THE NATION.
HELPING FIGHT OUR NATION'S
BIGGEST HEALTH CRISES FROM THE
OPIOID EPIDEMIC TO RARE DISEASE.
THAT'S NOT GETTING MESSAGED.
AND OUTSIDE STAKEHOLDER
ENVIRONMENT, DON'T UNDERSTAND
WHAT'S HAPPENING.
FROM MY UNDERSTANDING, ALL OF
THAT'S HAPPENING WITH ABOUT --
WITHIN 32% OF THE NCATS BUDGET.
THAT'S NOT PART OF THE PROGRAM.
AS YOU'RE WORKING ON THE
STRATEGIC PLAN, SPEND A LOT OF
TIME ON OPTIMIZING IMPACT AND
INFLUENCE AND PULLING US IN.
>>COMMUNICATING.
>> UH-HUH.
>> FANTASTIC.
THANK YOU.
I WROTE DOWN QUITE A LOT ON
THAT.
THANK YOU.
SERGIO?
>> YES, I'M LOOKING AT THE
STRATEGIC PLANNING PROCESS, AND
IT'S A VERY TOUGH PROCESS, BY
THE WAY.
STAKEHOLDER ENGAGEMENT AND
LOOKING AT THE WONDERFUL IDEA OF
HAVING THE RFI TO RECEIVE
INFORMATION.
WHEN YOU SAY GATHER FEEDBACK,
THAT'S WHEN YOU START OUTLINING
AND DRAFTING THE PLAN.
CAN YOU SPEAK MORE ABOUT THAT?
BECAUSE THERE ARE GREAT
OPPORTUNITIES THERE TO NOT ONLY
IDENTIFY GROUPS THAT HAVE
PROVIDED INPUT, YOU GO BACK TO
THOSE PEOPLE AND LET THEM KNOW
THIS IS WHAT WE HEARD.
YOU HAVE FURTHER COMMENTS, AND
ANYTHING ELSE YOU CAN THINK OF,
NOW YOU'RE THINKING OF.
SO YOU PROVIDE FEEDBACK ON THE
FEEDBACK THAT YOU GET.
AND THE OPPORTUNITY THAT I SEE
THERE IS THAT YOU CAN HAVE A
GROUP THAT CAN BE YOUR
DISSEMINATION AGENTS.
SO, I THINK CREATING THOSE LIFTS
OF NOT ONLY THOSE THAT YOU
ALREADY HAVE BEEN WORKING WITH
BUT THE IDEA HERE LIKE THINGS I
HEARD AS WELL, TAPPING INTO
INDUSTRY, OTHER GROUPS, SO WOULD
BE VERY HELPFUL, AT LEAST FOR
ME, IF YOU HAVE IN TERMS OF
TIMELINES, PLANNING PROCESS, YOU
CAN BE FAT A LITTLE BIT MORE AND
INCLUDE DISSEMINATION THERE IN A
WAY THAT YOU WILL ONCE AGAIN
MAXIMIZE, OPTIMIZE, YOU KNOW,
STRATEGIC PLAN.
A STRATEGIC PLAN AS WELL.
>> I THINK JONI CALLS THIS A
CO-DESIGN.
WE'RE ONE PART OF IT AND YOU ARE
THE OTHER PART.
I THINK WE CALL THIS AN UPDATE.
COMING BACK TO YOU AT THE NEXT
COUNCIL I'M SURE.
THIS IS A LONG PROCESS BECAUSE
IT REALLY HELPS US REALLY
DEVELOP SOME PRIORITIES FOR
MOVING FORWARD OVER THE NEXT 5
TO 10 YEARS AND IT'S REALLY --
YOU'RE GOING TO BE A BIG PART OF
MAKING SURE THAT WE GET THIS
RIGHT.
WHEN WE DESIGNED OUR FIRST PLAN
IN 2016 NCATS WAS SO NEW WE WERE
TRYING TO ESTABLISH OUR IDENTITY
WITHIN THE NIH.
AND ALSO TO REALLY CONNECT WITH
NOT ONLY THE SCIENTIFIC
COMMUNITY BUT REALLY THE NIH
COMMUNITY AS WELL.
AND I THINK THAT FIRST PLAN
INTERNALLY I THINK WE ALL FELT
LIKE IT FELT -- WE CALL IT
NCATS-IAN, FELT LIKE US, WHEN WE
HAD THAT WORD CLOUD DURING THE
PRESENTATION, THAT'S HOW WE
FELT.
WE DID NOT KNOW IF THAT'S HOW WE
LOOKED EXTERNALLY.
SO I THINK THIS COMMUNICATION
AND THIS KIND OF ITERATIVE
PROCESS OF WORKING WITH YOU,
GOING TO THE NEXT STEPS, SAYING
ARE WE STILL -- THIS IS WHAT WE
THINK IS RIGHT, DOES IT LOOK
RIGHT TO YOU AND THAT WE'RE ON
TARGET, BUT I HEAR YOU LOUD AND
CLEAR.
I THINK ALL OF YOU.
ABOUT THE DISSEMINATION AND THE
OUTREACH AND COMMUNICATION AS WE
GO.
>> ONE LAST THING, AND, JONI,
YOU SAID SOMETHING THAT BROUGHT,
YOU KNOW, RESONATED VERY MUCH.
THAT IS THAT WORKING WITH AHRQ
AND OTHER AGENCIES TO LEVERAGE
AND SYNERGIZE WHAT CAN BE
SYNERGIZED, YOU KNOW, IS A GREAT
IDEA.
I WOULD LOVE TO SEE SOMETHING
LIKE THAT MORE PURPOSELY, SOME
OF THAT COORDINATION AND
INSPIRATION AS WELL BECAUSE, YOU
KNOW, YOU CANNOT DO EVERYTHING,
AS YOU MENTIONED.
BUT MAYBE THROUGH OTHER
INSTITUTES, FOR EXAMPLE, FOR
ALL, I'M THINKING ON THE
NATIONAL INSTITUTE FOR MENTAL
HEALTH, HEALTH DISPARITIES, FOR
EXAMPLE, AS WELL AS OTHER
INSTITUTES THAT CAN HELP WITH
THAT THREE-PART TYPE VISION YOU
HAVE, WONDERFUL VISION.
>> THANK YOU.
I AGREE.
WE DO HAVE OPPORTUNITIES EXPLORE
THAT.
WHERE WE HAVEN'T DONE THAT QUITE
YET.
SO I THINK WE HAVE STARTED
CONVERSATIONS AND THEY ARE
REALLY ABOUT SORT OF LEARNING
ABOUT ONE ANOTHER, GETTING TO
KNOW ONE ANOTHER, AND WE CAN
TAKE IT TO THE NEXT STEP HELPING
US ENGAGE IN THE STRATEGIC
PLANNING PROCESS.
I SEE THAT AS REALLY MORE OF AN
ITERATIVE PROCESS TOO WITH THOSE
KINDS OF GROUPS.
THE OTHER THING I WANT TO SAY,
I'LL LET MEREDITH CHIME IN, IN
TERMS OF THE FEEDBACK TO THE
FEEDBACK, I THINK PART OF THAT
WILL BE IN TERMS OF PROCESS FOR
US, THE INITIAL PIECE OF THAT
WILL PROBABLY BE IN THAT RFI AND
THAT WILL GO OUT.
THAT'S GOING TO BE OUR FIRST
STAB AT DISTILLATION OF THE
INPUT WE RECEIVE.
IN A WAY WE'LL BE ABLE TO
DISTILL THAT IN THE RFI, FEED
THAT BACK TO THE COMMUNITY AND
SAY, HERE, THIS IS WHAT WE'VE
HEARD, DID WE GET IT RIGHT?
WHAT PARTS DID WE GET RIGHT AND
WRONG?
AND NOW THAT YOU'VE HAD A CHANCE
TO THINK ABOUT IT WHAT ELSE ARE
YOU THINKING ABOUT?
IT WILL KIND OF COVER THE GAMUT
OF THOSE WAYS OF PROVIDING THE
FEEDBACK BACK TO THEM AND THEN
ASKING THEM, YOU KNOW, HOW DID
WE DO.
SO I'M HOPING THERE WILL BE SOME
OF THAT BUILT INTO THE -- EVEN
IN THIS EARLY PHASE OF THE RFI
PIECE AND WE CAN PERHAPS EVEN
CONTINUE TO DO SOME OF THOSE KEY
ENGAGEMENT ALSO WHERE IT MAKES
SENSE WHERE MAYBE WE WEREN'T
QUITE SPOT ON AND GO BACK AND
MAKE SURE THAT WE'RE
UNDERSTANDING WHERE WE CAN MAKE
THAT PROGRESS.
SO THOSE ARE SOME OF THE
STRATEGIES THAT WE'LL DO OR
TACTICS I SHOULD SAY FOR THE
STRATEGIC PLAN.
MEREDITH, DID YOU HAVE SOMETHING
TO ADD AND I'LL GO TO ANIKA?
>>YEAH, I'LL ADD QUICKLY, I
ECHO EVERYTHING YOU AND PENNY
SAID.
A COUPLE THINGS, MY PERMANENT
APPROACH TO THIS TYPE OF
ACTIVITY IS VERY MUCH AN
ITERATIVE AND FEEDBACK LOOPS.
AND IT REALLY DOES HAVE TO BE
BIDIRECTIONAL, NOT JUST US
TELLING YOU AND OTHER
STAKEHOLDERS BUT TAKING UP THE
FEEDBACK.
THAT'S SOMETHING, ALTHOUGH IT'S
HARD TO PUT IT IN A CHEVRON, YOU
KNOW, ON A SLIDE, IS VERY MUCH
AT THE CORE OF HOW WE THINK
ABOUT AND ARE APPROACHING THE
STRATEGIC PLAN.
IN TERMS OF TACTICS LIKE YOU
SAID, JONI, YOU KNOW, WE WEREN'T
SURE WHEN WE STARTED THIS AND WE
WANTED TO STAY OPEN MINDED, AND
SORT OF OPEN TO THE PROCESS,
PLAYING OUT IN FRONT OF US
INSTEAD OF PRE-DECIDING A PATH.
WE WEREN'T SURE WHAT THE RFI WAS
GOING TO LOOK LIKE, WHETHER MORE
QUESTIONS, BASED ON THE
ROUNDTABLE, AND WE WANTED TO
PROBE THAT.
WHAT PENNEY AND I AND THE TEAM
ARE THINKING NOW IS WE'VE GOTTEN
SUCH RICHNESS FROM ALL OF THESE
ROUNDTABLES AND OTHER MEETINGS
THAT WE PROBABLY HAVE A FAIR
AMOUNT THAT WE CAN THEN REFLECT
BACK IN THE RFI TO START KIND OF
GOING TO THAT NEXT LEVEL OF
DEPTH AND SAYING, OKAY, THIS IS
WHAT WE HEARD, NOW WHAT ARE WE
MISSING, WHAT DOES THIS LOOK
LIKE AS IT'S STARRING TO HONE.
THE OTHER PIECE, SERGIO, TO THE
POINT YOU WERE MAKING ABOUT
ENGAGING WITH THE OTHER I.C.s,
THERE'S SOME PIECES OF THAT THAT
HAPPENED ORGANICALLY BECAUSE THE
OTHER I.C.s ARE PART OF THE
PROCESS WHEN AN I.C. BUILDS A
STRATEGIC PLAN.
SO, THEY ACTUALLY ALL GET TO SEE
THE STRATEGIC PLAN, THEY WILL BE
PART OF THE LOOP IN.
ACTUALLY SOME OF THEM JOINED OUR
ROUNDTABLES, WE WERE PLEASED BY
THAT.
BUT THEY ALL DEFINITELY WILL BE
IN THE LOOP AND I HAVE FOUND,
AND I THINK, JONI, YOU WERE
PROBABLY -- IT WAS VERY EARLY
DAYS SO YOU WERE DEPUTY, YOU AND
I CAME ON BOARD ABOUT THE SAME
TIME, WOMEN'S HEALTH WAS DOING
THEIR STRATEGIC PLAN AND ENGAGED
US AND MIKE KURILLA AND I WORKED
WITH THEM ON THEIR PLAN.
SO, THERE ARE SOME OF THOSE
SYNERGIES BUILT INTO THE PROCESS
THAT WILL I THINK HELP US ALONG
THE WAY, YOU KNOW, FOSTERING
MORE RELATIONSHIPS BUT THERE'S A
LOT OF EXISTING RELATIONSHIPS
AMONGST OUR STAFF AND ACROSS
I.C.s THAT WILL BE HELPFUL AS
WE MOVE FORWARD IN SHAPING THE
PLAN.
THAT'S EVERYTHING.
>>THANK YOU FOR THAT.
ANIKA?
>> YEAH, I'M NOT SURE, IT WILL
BE INTERESTING TO SEE, I MEAN,
THE FIRST STRATEGIC PLAN READING
THROUGH IT, YOU KNOW, A LOT OF
WHAT YOU'RE DOING NOW IS IN LIE
AND YOU'VE DONE GREAT WORK SINCE
INCEPTION OF NCATS SO I'M NOT
SURE HOW MUCH YOU WANT WANT TO,
ESPECIALLY GIVEN THAT YOU LAID
ALL THESE GREAT PROGRAMS OUT BUT
I THINK TAKING THE RISK IS
IMPORTANT AND MAYBE THE WAY TO
BALANCE THAT IS BECAUSE OF THE
GREAT WORK YOU'VE DONE, IT GOES
BACK TO THE SAME THING, ENGAGING
PEEL, YOU'VE GOT GREAT IDEAS.
WE'VE CREATED ALL THESE
ACHIEVEMENTS FROM THE FIRST PLAN
BUT NOW WE SEE WHERE THERE'S
GAPS, MAYBE OTHER STAKEHOLDERS
WE HAVEN'T ENGAGED IN.
VENTURE CAPITALISTS, MORE
PRIVATE SECTOR, OTHERS, WILL
MANY INFORM YOU HOW DO WE TAKE
THIS FIRST STRATEGIC PLAN TO THE
NEXT LEVEL THAT IS MORE RISKY.
I ALSO HAVE THE SAME THOUGHT HOW
DO YOU BUILD THE STRATEGIC PLAN
NOW THAT ARPA-H IS CREATED SO
THAT'S GOING TO HAVE TO FACTOR
IN SO PROBABLY ON THE LIST YOU
TALKED ABOUT I.C.s, MIGHT BE
HELPFUL TO TALK TO HOW DOES
DoD INTERFACE WITH DARPA, AND
THEY DO A LOT OF PRODUCT
DEVELOPMENT AND MAY HAVE THINGS
TO GAIN, SAME WITH DEPARTMENT OF
ENERGY, THOSE COULD BE OTHER
PEOPLE YOU TALK TO AS WELL.
YOU HAVE GREAT FOUNDATION TO
BUILD UPON SO IT'S GOING TO BE
TOUGH TO PIVOT.
I DON'T KNOW IF YOU WANT TO DO A
FULL PIVOT OR INCREMENTAL
ADVANCES ON WHAT YOU'VE DONE
ALREADY.
IT WILL BE INTERESTING TO SEE.
>> YEAH, I THINK THAT THERE ARE
GOING TO BE ASPECTS WHERE WE'RE
GOING TO GO DEEPER BECAUSE NOW
WE KNOW MORE.
>> RIGHT.
>>WE HAVE REALLY LEARNED A LOT.
WE'VE LEARNED A LOT ABOUT DATA
SCIENCE.
WITH THE INFORMATICS, SDPI AND
THOSE THINGS.
GENE-TARGETED THERAPIES IS
SOMETHING WE DIDN'T HAVE A WHOLE
LOT OF IN THE FIRST PLAN.
AND TALKING ABOUT WHAT ARE THE
PRIORITIES, YOU KNOW.
I SEE IT THAT WAY BUT, AGAIN, WE
DID HAVE AND DID RECEIVE A LOT
OF COMMENTS ABOUT OTHER AREAS OR
OPPORTUNITIES TO ADDRESS.
>> IF YOU DON'T MIND, I'LL ADD
TO TO WHAT PEPPY WAS SAYING.
I PUT THIS IN THE CHAT.
WE'RE NOT IGNORING THAT PLAN IN
ANY WAY, SHAPE, OR FORM.
YOU KNOW, I THINK IT WAS DR.
RUTTER'S PURVIEW AS NEW
DIRECTOR, WANTED TO PUT HER
STAMP AND HER VISION ON THE PLAN
BUT I THINK FIRST OF ALL WE DO
WANT TO REFLECT BACK, THAT'S
EXPECTED AS TO WHERE DO WE COME
FROM AND WHAT HAVE WE DONE,
THERE'S ALSO AS YOU NOTED A LOT
OF RICHNESS IN THAT PLAN THAT WE
CAN BUILD ON.
AND SO WE'RE GOING TO BE LOOKING
AND FACTORING THAT INTO OUR
ANALYSIS AND SYNTHESIS OF THE
FEEDBACK AS WELL.
SO DEFINITELY WANT TO MAKE SURE
FOLKS DIDN'T THINK WE WERE
WORKING AWAY FROM THAT PLAN IN
ANY WAY, SHAPE, OR FORM.
>> BUILDING ON IT.
FIRM FOUNDATION.
>> THAT'S GREAT.
>> THANK YOU.
ANY OTHER COMMENTS?
OKAY.
I THINK WE'RE GETTING -- WE
MANAGED TO GO THROUGH TILL 5:30,
SO ALWAYS IMPRESSIVE.
I REALLY APPRECIATE YOUR
ENGAGEMENT.
I APPRECIATE YOUR ENGAGEMENT,
APPRECIATE YOUR INPUT,
APPRECIATE YOUR PARTNERSHIP.
I THINK THIS DISCUSSION HAS BEEN
INCREDIBLE.
>> I DON'T THINK WE'VE SAID
THANK YOU TO EVERYBODY.
NICE TO SEE EVERYBODY IN PERSON.
AT THE END OF THE DAY WHAT
YOU'RE PRESENTING IS THAT YOU'RE
SAVING AND CHANGING LIVES SO I
KNOW THESE ARE TECHNICAL
PRESENTATIONS BUT JUST THANK YOU
FOR THE WORK YOU DO.
IT'S BREATHTAKING.
>> EXTRAORDINARY, CONSISTENTLY,
IT'S AMAZING WE'RE EVEN VOTING
ON THIS.
KEEP DOING GREAT WORK.
[CROSSTALK]
>> IMPACTING SO THANK YOU.
>>THANK YOU VERY MUCH FOR THOSE
COMMENTS.
HEARTFELT, SO THANK YOU.
AND WE WON'T TAKE A VOTE ON
THAT.
I'LL JUST TAKE IT AS
APPRECIATION.
[LAUGHTER]
NEXT TIME I PROMISE TO BE THERE
IN PERSON, FOR CRYING OUT LOUD.
THIS HAS BEEN -- I ADMIT TO
HAVING A BIT OF FOMO, TEXTING
PEOPLE ALONG THE WAY, HOW IS
EVERYTHING GOING, AND NOT TO BE
CONFUSED WITH NOFO, THIS IS
FOMO.
[LAUGHTER]
>> JONI, ALSO, TO THANK ANNA
AND YOUR FULL TEAM.
[APPLAUSE]
>> YES, THANK YOU.
IT IS A PROCESS, I TELL YOU, TO
PULL THESE THING TOGETHER.
THEY DO A FABULOUS JOB FOR THE
WHOLE COUNCIL TEAM AND THE
REVIEW BRANCH AS WELL.
IT'S JUST REALLY IMPRESSIVE HOW
MUCH WORK IT IS, AND I
APPRECIATE ALL OF THAT.
SO I LOOK FORWARD TO SEEING YOU
NEXT TIME AND, AGAIN, WE
PROBABLY MIGHT REACH OUT TO YOU
IN BETWEEN NOW AND THEN AS WE
CONTINUE TO THINK ABOUT THESE
QUESTIONS AND ISSUES.
BUT I APPRECIATE ALL OF THE
FEEDBACK.
IN DUE COURSE AS WE'RE CLOSING
OUT THIS SESSION, WE WILL GO
AHEAD AND GAVEL OURSELVES OUT SO
I'M GOING TO TURN THIS OVER.
>> THIS MAY BE THE LAST TIME I
GET TO DO THIS.
THANKS, EVERYBODY.