I'LL BEGIN BY GAVELLING US INTO
THE 32nd NCATS COUNCIL.
WELCOME, EVERYBODY.
IT'S ALWAYS VERY AWKWARD TO DO
THIS VIRTUALLY.
I ONLY HAVE ONE COMPUTER AND ONE
CAMERA SO ALWAYS A LITTLE BIT
AWKWARD BUT NICE TO SEE YOU.
THANK YOU FOR JOINING OUR 32nd
MEETING FOR THE NATIONAL CENTER
FOR ADVANCING TRANSLATIONAL
SCIENCES ADVISORY COUNCIL.
IT'S AN EXCITING DAY AND I LOOK
FORWARD TO SAYING A BIT MORE IN
THE COMMENTS DURING THE OPEN
COUNCIL SESSION.
BEFORE I DO, AS I WELCOME YOU, I
WANT TO TURN IT OVER TO ANNA TO
KICK US OFF WITH MAKING SURE WE
DO ROLL CALL AND A FEW OTHER
THINGS.
ANNA, OVER TO YOU.
>>THANK YOU, JONI.
I'M GOING TO ASK FOR MEMBERS TO
SAY PRESENT WHEN THEIR NAMES ARE
CALLED.
DR. SERGIO
[ROLL CALL]
>>WE HAVE NO MEMBERS
PARTICIPATING IN AN AD HOC
CAPACITY TODAY.
WE'LL HAVE A FEW ANNOUNCEMENTS
FOLLOWED BY A DIRECTOR'S REPORT
AND HAVE ONE CONCEPT AND HAVE
THE DIRECTOR OF THE NIH
EXTRAMURAL REPORT AND UPDATE
FROM THE OFFICE OF SPECIAL
INITIATIVES AND THEN WE'LL
ADJOURN FOR THE DAY.
EVERYONE IS A PANELIST SO YOU'RE
FREE TO SPEAK BUT WE REQUEST YOU
USE THE RAISE HAND FEATURE AND
WAIT TO BE CALLED UPON TO
PROVIDE INPUT AND ALLOW
PRESENTERS TO MOVE THROUGH THEIR
COMMENTS.
WE'RE USING THE VIDEOCAST
FEATURE TODAY.
AUDIENCE MEMBERS MAY SUBMIT
COMMENTS UNTIL FEBRUARY 10 USING
THE SEND LIVE FEEDBACK BUTTON OR
SEND AN E-MAIL TO THE NCATS
COUNCIL INPUT MAILBOX.
MAKE SURE WHEN YOU ARE SPEAKING
YOUR CAMERA IS ON SO YOU ARE
SPOTLIGHTED.
IF YOU'RE NOT ON CAMERA THE
PREVIOUS VIEWER REMAINS
SPOTLIGHTED.
THE FUTURE DATES ARE MEETINGS IN
2023 ARE MAY 25 AND SEPTEMBER
28.
IN 2024, THE JANUARY MEETING
WILL BE ON THE 18th AND 19th AND
THAT WILL WILL BE A VIRTUAL
MEETING AND THEN REGULAR FACE TO
FACE IN-PERSON MEETINGS ON MAY
23 AND SEPTEMBER 26.
AND COULD HAVE A MOTION TO
APPROVE THE MINUTES FOR THE
SEPTEMBER 2022 MEETING?
>>SO MOVED.
>>SECOND.
ALL IN FAVOR?
ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT THE MINUTES ARE
APPROVED.
SERGIO?
>>I ABSTAIN BECAUSE I WASN'T IN
THE MEETING.
>>THANK YOU.
SO NOW WE ARE ASKING FOR
APPROVAL OF THE NCATS GENERAL
OPERATING PROCEDURES FOR 2023?
WE'RE SEEKING YOUR APPROVAL.
DO WE HAVE A MOTION TO APPROVE
THE GENERAL OPERATING PROCEDURES
FOR 2023?
>>SO MOVED.
>>SECOND.
>>ANY OPPOSED?
ANY ABSTENTIONS?
THE 2023 GENERAL OPERATING
PROCEDURES FOR THE NCATS
ADVISORY COUNCIL HAS BEEN
APPROVED.
BACK FOR THE DIRECTOR'S REPORT.
THANK YOU.
>>OKAY.
THANK YOU SO MUCH ANNA I'M GOING
TO DO A QUICK CHECK TO MAKE SURE
I HAVE CONTROL OF THE OF THE
MOVEMENT OF THE SLIDES.
THANK YOU, ANNA.
I'M EXCITED TO PRESENT THE ROLL
TO COUNCIL NOW AND I'LL HAVE QR
CODES THROUGHOUT AND IN CASE IF
YOU'RE LOOKING AT THE OTHER
CONTENT YOU'LL BE PRESENTING
YOU'LL HAVE A GENERAL OVERVIEW
BUT IF YOU'D LIKE A DEEP DIVE ON
ANYTHING WE'LL HAVE QR CODES TO
DO THAT.
I'LL GET STARTED WITH JUST
KICKING OFF FIRST WITH SOME OF
THE COMINGS AND GOINGS AT NCATS
AND I BECAME THE DIRECTOR AND
IT'S AN HONOR TO HAVE THE ROLE
AND HAVING DONE IT IN AN ACTING
CAPACITY I HAD THE PRIVILEGE AND
HONOR OF WORKING WITH THE
COUNCIL MEMBERS AND ALL THE
NCATS STAFF IN THE CAPACITY FOR
A BIT AND WHAT A GROUP.
I THINK THIS IS VERY EXCITING TO
MOVE FORWARD WITH THE STAFF AT
NCATS AND OUR COUNCIL MEMBERS
AND LOOKING FORWARD TO WORKING
WITH YOU IN THIS CAPACITY AND
THERE'LL BE MORE TO COME AS THE
YEAR PROGRESSES.
JUST A TEASER FOR NOW.
IT'S ALSO EXCITING BECAUSE WE
HAVE NEW COUNCIL MEMBERS, SERGIO
AGUILAR-GAXIOLA IS AN OLD FRIEND
OF MINE AND DID WORK ON
ADDICTION AND EXCITED TO SEE
HIM.
ROBIN MERMELSTEIN AND EXCITED TO
HAVE THE TWO VOICES ON OUR
COUNCIL.
ANNICA WAYMAN WE'VE DONE WORK ON
TRAINING AND CAREER DEVELOPMENT
TO THE UNIVERSITY OF SHADY GROVE
AND SHE'S DOING A TREMENDOUS JOB
THERE.
IT'S EXCITING TO HAVE HER ON OUR
COUNCIL TOO.
WELCOME A BOBOARD AND WE'VE HAD
ANDREW LOWE.
IT'S THEIR LAST COUNCIL AND
THEY'VE BEEN HUGE CONTRIBUTORS
OVER WHAT WE'VE BEEN DOING THE
LAST FEW YEARS SERVING ON
COMMITTEES AND THINKING OF
THINGS RELATED TO RETURN ON
INVESTMENT BUT WE DON'T CALL IT
THAT.
THINGS RELATED TO DERISKING AND
HOW WE CAN FIND AN IMPACT ON THE
ECONOMY THROUGH THE WORK WE DO.
I APPRECIATE YOUR GUIDANCE AND
ADVICE ALONG THE WAY.
THANK YOU SO MUCH TO TED AND
ANDREW.
CONGRATULATIONS ON YOUR TIME
WELL SERVED.
AND WE'RE ALSO STARTING TO BUILD
UP OUR CAPACITY FOR OUR REVIEW
BOARD.
AN EXCITING MOMENT FOR US AND
VERY EXCITED TO HAVE SIMON FROST
JOIN US ON THE REVIEW BOARD.
WE'RE GETTING CLOSER AND CLOSER
TO HAVE THEM AND EXCITED TO SEE
THAT COME TOGETHER AND MORE
INFORMATION WILL BE COMING ONCE
THAT HAPPENS.
LASTLY -- ACTUALLY, I WANT TO
MAKE SURE I MENTION -- I DON'T
KNOW I MENTIONED SIMON'S, SIMON
FROST'S NAME AND HE HAS AN
IMPORTANT VOICE FOR THE RARE
DISEASE GROUP AND EXCITED TO
HAVE HIM AT THE TABLE FOR THE
REVIEW BOARD.
SO THEN THERE'S THE GROUP HERE
ON THE REVIEW BOARD ALSO LEAVING
US AND ANDREW AND TED AND KAREN
AND LIZ HAVE BEEN LONG STANDING
MEMBERS ON THE REVIEW BOARD.
THEY HAVE REALLY PULLED TOGETHER
BEFORE WE WENT ON A BRIEF HIATUS
FOR THE QUORUM, THE REVIEW BOARD
PULLED TOGETHER REPORTS FOR US
TO CONSIDER AND ONCE THE GROUP
RECONVENES I'M EXCITED TO SEE
THE PROCESS AND SAD TO SEE THE
CAN REVIEW BOARD MEMBERS TAKE A
FAREWELL BUT WE MAY REACH OUT
AGAIN.
PLEASE STAY TUNED AND CERTAINLY
YOU'RE ALWAYS FRIENDS OF NCATS.
I ALSO WANTED TO SAY TOO THAT
LILLY PORTILLA IS TAKING OVER AT
NOVAX.
SHE HAS BEEN WITH NCATS SINCE
THE BEGINNING AND ESTABLISHED
THE OFFICE OF STRATEGIC
ALLIANCES AND THE WORK WITH THE
SPIR AND STTR PROGRAMS AND TECH
TRANSFER PROGRAMS.
SHE'S BUILT UP THE PROGRAM AT
NCATS AND AN INCREDIBLE GROUP
AND BALKI WILL BE IN THE ROLE
MOVING FORWARD.
AS MANY WHO HAVE KNOWN HIM HE'LL
STEP IN AND IT WILL BE A
SEAMLESS TRANSITION AND I'M
LOOKING FORWARD TO WORKING WITH
HIM IN THE ACTING ROLE UNTIL WE
CAN FILL THIS ROLE.
SOME SOONER THAN OTHERS
DEPENDING ON WHERE THEY ARE IN
THE PROCESS.
GIVEN NOW IS THE PERMANENT
DIRECTOR I'M ABLE TO MOVE
FORWARD ON FILLING OTHER
POSITIONS.
IT'S RELEASED THAT HOLD, IF YOU
WILL, WE'RE EXCITED TO BE ABLE
TO OPEN UP THE DEPUTY DIRECTOR
ROLE.
WE'RE ESTABLISHING A CHIEF
DIVERSITY OFFICE AND LOOKING
FORWARD FOR THE OFFICE OF
STRATEGIC ALLIANCES AND WE'LL
ALSO BE OPENING UP THE SEARCH
FOR THE DIVISION OF PRE-CLINICAL
INNOVATION AND ANOTHER SEARCH
THAT'S ONGOING IS THE DIVISION
OF RARE DISEASE RESEARCH
INNOVATION DIRECTOR.
A VARIETY OF POSITIONS TO BE
FILLED AND I THINK THIS WILL
HELP US STAND ON MUCH MORE SOLID
FOUNDATION ONCE THE POSITIONS
ARE FILLED.
THEY'LL BE AT FULL CAST AND BE
ABLE TO HIT THE GROUND RUNNING
WHEN THAT'S THERE.
WE LOOK FORWARD TO MOVING AND
WORK IN THE SPACE OVER THE
COMING YEARS -- SORRY, YEAR.
I HOPE IT WON'T TAKE YEARS AND I
HOPE WE'LL HAVE THAT IN PLACE
FAIRLY SOON.
ON A SAD NOTE NCATS HAS
EXPERIENCED THE LOSS OF A COUPLE
COLLEAGUES RECENTLY AND WANT TO
TAKE A MOMENT TO RECOGNIZE THOSE
COLLEAGUES.
PAUL SHINN WAS THE DPI COMPOUND
MANAGEMENT GROUP LEADER AND
PLAYED A ROLE AND CAN THE WORK
AT NCATS AND HAS ENABLED A
VARIETY OF WORK TO DO
COMBINATION COMPOUND SCREENING
AT NCATS AND HE'S BEEN ABLE TO
LEAD THE WORK THAT HE'S BEEN
DOING HAS BEEN ABLE TO LEAD TO A
VARIETY OF GROUPS BEING ABLE TO
DO CANCER CLINICAL TRIALS.
SO HIS WORK HAS BEEN TRULY
INCREDIBLE AND HE WILL BE VERY
MUCH MISSED WITHIN NCATS.
SIMILARLY, JACOB MENSAH HAS
BUILT THE BACK END OF NCATS AND
PULLED THAT ALTOGETHER.
WE'RE VERY SAD ABOUT THE LOSS OF
OUR COLLEAGUES.
I WANTED TO TAKE A MOMENT TO
MENTION THEM AND RECOGNIZE THEIR
WORK AND THEIR CONTRIBUTIONS TO
NCATS.
IN TERMS OF NEWS AND
ANNOUNCEMENTS I WANTED TO START
WITH SOME THINGS HAPPENING
AROUND NIH.
I'LL TALK OF SOME THINGS GOING
ON AT NCATS.
FIRST OF ALL, WHAT WILL BE
HAPPENING AT 3:00 TODAY, MIKE
LAUER THE DEPUTY DIRECTOR FOR
EXTRAMURAL RESEARCH WILL TALK
ABOUT THE NIH DATA MANAGEMENT
SHARING POLICY AND IMPLEM
IMPLEMENT
IMPLEMENTATION.
IT HAD A DATE OF JANUARY 25,
2023 WAS THE IMPLEMENTATION
DATE, YESTERDAY, THE POLICY IS
NOW IMPLEMENTED.
MIKE WILL HIGHLIGHT THE NEW
REQUIREMENTS AND RESOURCES TO
NAVIGATE THIS NEW POLICY.
SO PLEASE STAY TUNED FOR HIS
TALK.
THERE'S BEEN A COUPLE THINGS I'D
LIKE TO MENTION TOO AROUND OTHER
AREAS THAT NIH HAS BEEN WORKING
ON THROUGH THE ADVISORY
COMMITTEE OF THE DIRECTOR.
THEIR MEETING HAPPENED DECEMBER
8 AND 9 AND THERE WERE A FEW
PROPOSALS I THOUGHT WERE
IMPORTANT TO BRING TO YOUR
ATTENTION MAINLY FOR AWARENESS
BUT ONE IN PARTICULAR I'LL
HIGHLIGHT IN A MOMENT THAT I
THINK YOU MIGHT BE EVEN MORE
INTERESTED IN.
THIS ONE I JUST WANTED TO
HIGHLIGHT IS A PROPOSED NEW
FRAMEWORK FOR NIH PEER REVIEW
CRITERIA.
THERE'S A REQUEST FOR
INFORMATION OUT ON THE STREET
NOW AND IT WILL BE OPEN UNTIL
MARCH 2023.
IF YOU GET YOUR PHONE OUT YOU
CAN TAKE A LOOK AT THE QR CODE
AND DOWNLOAD THAT TO BE ABLE TO
FIND MORE INFORMATION.
ESSENTIALLY, THIS IS A WAY TO
REFOCUS THE FIRST LEVEL OF PEER
REVIEW.
THE COUNCIL DOES THE SECOND
LEVEL OF PEER REVIEW AND FOCUS
THE FIRST LEVEL OF PEER REVIEW
AND STREAMLINE IT TO REDUCE
BURDEN ON VIEWERS AND
INCENTIVIZE REVIEW AND HOPE IT
WILL MITIGATE THE BIAS IN THE
PROCESS LOOKING AT INVESTIGATOR
AND ENVIRONMENTS AND THE CONTEXT
OF THE PROPOSED RESEARCH AS
THINGS THAT CAN BE DISCUSSED BUT
NOT NECESSARILY SCORED.
SO PLEASE TAKE A LOOK AT THE
RFI.
IF YOU HAVE ANY THOUGHTS ON THAT
FEEL FREE TO WEIGH IN.
ANOTHER ADVISORY COMMITTEE IN
THE DIRECTOR TOPIC WAS IN
RE-ENVISIONING THE POST-DOCTORAL
TRAINING.
IT WILL BE A SUBCOMMITTEE OUT OF
THE ACD TO LOOK AT THE POSDOC
POSITION AND THERE'S A SHORTAGE
OF Ph.D.s WITHIN THE U.S. AND
SOME ARE GOING TO PERMANENT
POSITIONS.
THIS IS SOMETHING WE WANTED TO
EVALUATE MORE, UNDERSTAND WHAT'S
LEADING TO THAT AND INFLUENCING
THE FACTORS AND TO UNDERSTAND IF
THERE ARE WAYS IN WHICH WE CAN
INCREASE RETENTION INTO THE
Ph.D. POSTDOC WORKFORCE AREA AND
SUPPORT THE TRAINING FOR THE
POSDOCS OR PERHAPS CONSIDER
OTHER WAYS TO MANAGE THAT.
THESE ARE THINGS ON THE TABLE
FOR THE PARTICULAR WORKING GROUP
TO ESTABLISH.
WE EXPECT RECOMMENDATIONS COMING
OUT TOWARDS THE END OF YEAR AND
IF YOU'D LIKE MORE INFORMATION A
QR CODE IS HERE.
THE LAST ONE I WANTED TO MENTION
FROM THE ACD WORK GROUP IS ONE
ON CATALYZING THE DEVELOPMENT
AND USE OF ALTERNATIVE METHODS
TO ADVANCE BIOMEDICAL RESEARCH.
YOU RECOGNIZE THIS MORE DIRECTLY
AS SOME OF THE ACTIVITIES NCATS
HAS BEEN INVOLVED IN IN THINKING
ABOUT THE TISSUE CHIPS AND 3-D
BIO PRINTING AND OTHER
APPROACHES LIKE ARTIFICIAL
INTELLIGENCE AND THINGS OF THAT
NATURE AND THINK ABOUT HOW THE
ALTERNATIVES METHODS CAN BE
EXPANDED TO AND STRENGTHS AND
WEAKNESSES AND HOW TO INTEGRATE
THEM IN THE REGULATORY SPACE AND
WHERE ARE THEY BEST USED IN THE
CONDITIONS AND DISEASES OF KEY
INTEREST AND WHERE THEY MAY NOT
BE WELL USED.
THESE ARE AREAS WE WANT TO START
TO HIGHLIGHT AND INVEST MORE
FOCUSSED EFFORTS ON THIS AND
ATTENTION ON THIS SO WE CAN
DEVELOP MORE -- BETTER PLANS
MOVING FORWARD.
AGAIN, THIS WILL BE A WORKING
GROUP THAT WE'LL CONVENE
PROBABLY THE FINAL
RECOMMENDATIONS IN DECEMBER OF
THIS YEAR AND STAY TUNED ON THAT
AND HAPPY TO SAY TOO THAT DAN
TAGLY OUR OFFICE OF SPECIAL
INITIATIVES DIRECTOR WILL BE THE
EX-
EX -OFFICIO MEMBER.
AND ANOTHER NIH EFFORT IS
QUANTUM SENSING AND QUANTUM
INFORMATION SCIENCES AND I
CO-CHAIR THIS WITH SUSAN
GREGURICK AND THE NIMH.
THIS IS A COLLECTIVE GROUP AND
HAS A BROADER NUMBER OF PEOPLE
ACROSS NIH TO PLAY A ROLE IN THE
ACTIVITY TO EXPLORE THE
POTENTIAL OF SOME OF THE QUANTUM
WORK.
THERE WAS A WORKSHOP HELD
RECENTLY.
IT'S NOT QUITE UP ON VIDEOCAST
BUT SHOULD BE SHORTLY.
WHEN THAT HAPPENS I'M HAP --
REACH OUT AND I'M HAPPY TO SEND
YOU THAT.
A FEW MORE THINGS ON NCATS
SPECIFIC ACTIVITIES I WANTED TO
HIGHLIGHT IS THAT AS A NEW
DIRECT I HAVE THE OPPORTUNITY TO
TAKE A LOOK BACK AT OUR
FOUNDATION, OUR LAST 10 YEARS
AND TAKE A LOOK AT HOW WE CAN
BUILD ON THAT AND MOVE FORWARD
TOGETHER AND SO I'M LOOKING
FORWARD TO BUILDING A STRATEGIC
PLAN FOR THE NEXT 10 YEARS.
WE'RE WORKING ON THAT PROCESS
NOW AND IT WILL ESSENTIALLY BE
AROUND MY AUDACIOUS GOALS I KNOW
YOU HEARD ABOUT BEFORE SO I
WON'T DWELL ON THEM BUT THE IDEA
IS OUR VISION IS TO BRING MORE
TREATMENTS FOR ALL PEOPLE MORE
QUICKLY.
SO WORKING AROUND THOSE THREE
CONCEPTS AND PROBABLY A FEW MORE
AROUND DATA SCIENCE, TRAINING
AND MENTORING AND THINGS OF THAT
NATURE WE WILL INCLUDE THOSE
ACTIVITIES AS WELL BUT RIGHT NOW
WE WANT TO GET ORGANIZED AROUND
THIS AND BUILD A PLAN FOR
STAKEHOLDER AND I THINK IT'S
IMPORTANT TO DO A LOT OF
LISTENING AT THIS STAGE AND
UNDERSTAND WHERE WE CAN FIND
STRENGTHS IN OUR BROAD ECO
SYSTEM OF WHAT WE'RE TRYING TO
DO IN THE SPACE OF BRINGING MORE
TREATMENTS TO ALL PEOPLE MORE
QUICKLY.
PLAY TO THE STRENGTHS AND HOW
CAN WE AT NCATS ENABLE THAT AND
DEVELOP OUR PROGRAMS TO DO JUST
THAT.
WE'RE STARTING THAT NOW.
YOU'LL PROBABLY BE HEARING MORE
ABOUT THAT TOWARDS THE END OF 1
AND START OF Q2 THIS YEAR AND
STAY TUNED FOR ACTIVES AROUND
THIS BECAUSE I -- ACTIVITIES
BECAUSE I KNOW I'LL SEEK YOUR
INPUT AND THIS WILL BE A DEEPER
TOPIC WE'LL TALK ABOUT IN MAY AT
OUR COUNCIL MEETING THERE TOO.
STAY TUNED FOR THAT.
IT'S A BIT OF A TEASER FOR THE
STRATEGIC PLAN PROCESS WE'LL BE
DOING.
AND A SENSE OF THE BUDGET.
LAST TIME WE WERE IN A
CONTINUING RESOLUTION AND NCATS
RECEIVED AN INCREASE OF 3.8% FOR
$23 MILLION MORE AND IN THE NON
CTSA PROGRAM WE RECEIVED 6.6%
INCREASE.
WE'RE PRETTY EXCITED ABOUT THIS.
AND OVER ALL NIH RECEIVED $47.7
MILLION AND WE'RE EXCITED ABOUT
HOW THIS IS TURNING OUT AND HOPE
IT WILL CONTINUE BUT WE'RE LOOK
FORWARD TO ADVANCING MORE
SCIENCE THROUGH THESE
ACTIVITIES.
REALLY GREAT TO SEE THE SUPPORT.
I WANT TO TALK ABOUT A FEW
PROGRAM UPDATES AND START WITH
VIGNETTES ABOUT THE CTSA PROGRAM
AND ABOUT THE FALL CTSA MEETING
WHICH WAS IN-PERSON AFTER A
HIATUS DURING THE PANDEMIC.
WE'RE FINALLY ABLE TO MEET IN
PERSON WHICH IT FELT LIKE IT HAD
BEEN 10 YEARS OR MORE SINCE WE
MET IN PERSON.
IT HADN'T BEEN BUT THE
EXCITEMENT WAS IN THE AIR AND
THIS WAS ONE PARTICULARLY
FOCUSSED ON DIVERSITY, EQUITY,
INCLUSION AND ACCESSIBILITY TO
CARE AND INCREDIBLE DISCUSSIONS
FROM THAT PANELS THAT WERE
PRESENTED.
AND A KEY NOTE ADDRESS WAS GIVEN
AS WELL.
EXCITED TO HAVE THIS GROUP BACK
IN ACTION AND I HOPE THAT WILL
CONTINUE AND THE PANDEMIC WILL
CONTINUE TO HOPEFULLY SUBSIDE.
AND SHORTLY AROUND THE TIME OF
THE FALL MEETING WE'VE THOUGHT
OF HOW TO GET MORE DIRECT
COMMUNICATION WITH THE CTSA
PROGRAM.
SO I PULLED TOGETHER A SERIES OF
ROUNDTABLES, LARGELY STARTING
WITH THE 60 CTSA PIs AND THEIR
EXECUTIVE LEADERSHIP.
THE WHOLE PURPOSE HERE TO COME
TOGETHER IN THIS ROUNDTABLE
FASHION WITH THE CTSA AND
LEADERSHIP IS TO FIND WAYS TO
DISCUSS WAYS TO PROMOTE
COMMUNICATION ACROSS THE
CONSORTIUM AND PROVIDE FEEDBACK
AND REQUEST FOR MORE
OPPORTUNITIES FOR DISCUSSION,
FRANKLY.
AND UNDERSTAND HOW TO MOVE MORE
STRATEGICALLY.
I'M SHOWING A WORD CLOUD.
WE ASKED THE GROUP WHAT WAS THE
MOST IMPORTANT WORK THE CTSA
DOES AND YOU SEE COLLABORATION,
TRAINING, SUPPORT NETWORK,
EDUCATE, INFORMATICS AND GREAT
TO SEE THE WORDS ABOUT
CONNECTION AND PULLING TOGETHER.
I'M LOOKING FORWARD TO
CONTINUING THIS ACTIVITY WITH
DIRECT COMMUNICATION WITH OUR
CTSA PROGRAM AND WORKING SIDE BY
SIDE WITH DR. MIKE CARULA WHICH
LEADS THE PROGRAM AND I THINK IT
WILL BE AN EXCITING TIME AND
HELPFUL FOR UNDERSTANDING HOW WE
CAN ENSURE THE CTSA AND OUR
NCATS STRATEGY PLANNING ARE
WORKING IN TANDEM AND
COMPLEMENTARY TO EACH OTHER.
IT'S AN EXCITING SERIES OF
THINGS WE'LL BE DOING AND WE'LL
CONTINUE THE ROUNDTABLES WITH A
VARIETY OF OTHER GROUPS WITHIN
THE CTSA PROGRAM AND PERHAPS USE
THEM FOR OTHER ACTIVITIES AS
WELL.
AND THIS IS WORK THAT CAME OUT
OF THE RECRUITMENT INNOVATION
CENTER LED BY SARA WILKENS.
WE HEARD A LOT OVER THE PANDEMIC
ABOUT THE ISSUE THAT TRUST IS
REALLY LOW IN BIOMEDICAL
RESEARCH AMONG MINORITIZED
RACIAL AND ETHNIC GROUP UNDER
REPRESENTED IN RESEARCH AND
THESE SAME GROUPS EXPERIENCED
WORSE HEALTH OUTCOMES.
WE NEED TO ADDRESS THIS MORE
DIRECTLY.
THIS GROUP HAS DEVELOPED A SCALE
TO HELP US DO JUST THAT.
THIS IS A NEW INSTRUMENT TO
UNDERSTAND HOW TO THINK ABOUT
TRUST AND DISTRUST WITH SPECIFIC
SCALES THAT ARE WELL VALIDATED
AND DEVELOPED.
SO WE'LL BE ABLE TO USE THIS
KIND OF SCALE IN COMMUNITIES AND
START TO TACKLE AREAS AROUND
TRUST TO START TO ENGAGE ON AND
HOPEFULLY START TO TRANSFORM.
ANOTHER VIGNETTE IS A
COLLABORATION ACROSS THE CTSA
PROGRAM AND THIS STUDY IN
PARTICULAR IS ABOUT CILIARY
DYSKINESIS AND THE ADULTS WITH
THE DISEASE HAD PROBLEMS WITH
REPRODUCTION.
A COLLABORATION HAPPENED WITH
THIS GROUP TO UNDERSTAND THE
MECHANISMS AND LOOK AT POTENTIAL
THERAPEUTIC.
DIS AND GETTING AT THE
TREATMENT APPROACH IS WHAT WE'RE
HOPING TO SEE.
AND WE FOUND OUT THAT THERE IS
ACTUALLY MORE WIDESPREAD ISSUES
AROUND SOIL FUNGI IN OTHER AREAS
OF THE UNITED STATES SO RAISING
AWARENESS AND SHOWING THIS IS
THE CASE FOR A VARIETY OF OTHER
REGIONS ACROSS THE COUNTRY WILL
HELP REDUCE DIAGNOSES FOR PEOPLE
IN THESE AREAS AND WHAT TO LOOK
FOR IN CASES FOR SYMPTOMS THAT
OVERLAP WITH THINGS SEEN WITH
YELLOW FEVER, SORRY, VALLEY
FEVER.
THIS IS AN IMPORTANT PIECE OF
INFORMATION AND WANT TO MAKE
SURE I RAISE THE AWARENESS FOR
THE GROUP.
AND I'M GOING TO CLOSE ON THIS
ONE.
THIS IS A COMMERCIALIZATION OF A
PRODUCT.
THIS IS REALLY HOW THE IDEA OF
SUTURING NEUROSURGERIES IS QUITE
TIME CONSUMING.
THIS GROUP OUT OF OREGON, THE
OREGON CLINICAL AND
TRANSLATIONAL RESEARCH INSTITUTE
CREATED A DEVICE TO ENABLE
SUTURES IN A FRACTION OF THE
TIME THAT TAKES OTHER WISE AND
ANOTHER ANOTHER DEVELOPMENT FOR
ENABLING HEALTH CARE AND
HOPEFULLY REDUCING CONCERNS
AROUND THE LENGTH OF SURGERIES
THAT HAPPEN AS WELL.
THIS IS A GREAT ADVANCEMENT TOO.
AND ACTUALLY I HAD ONE MORE
VIGNETTE.
I WANTED TO GIVE A SHOUT OUT TO
THE SMART IRBs.
NCATS HAS BEEN INVOLVED IN THE
EMERGENCY PREPAREDNESS AND THERE
WAS A WHITE HOUSE OFFICE OF
SCIENCE AND TECHNOLOGY REQUEST
FOR INFORMATION THAT CAME OUT
NOT TOO LONG AGO AND SMART IRB
WAS A WAY MENTIONED IN THINKING
OF ADOPTING FOR EMERGENCY MASTER
AGREEMENT FRAMEWORKS TO REALLY
HIGHLIGHT THE IDEA OF WHERE WE
CAN MOVE MORE QUICKLY WHEN
THERE'S URGENCY THERE.
I WANTED TO SHOW YOU THE SLIDE
BECAUSE THE GRAPHIC IN THE
CENTER SHOWS THE VALUE OF THE
SMART IRB.
THE NUMBER OF SITE APPROVALS
SHOWN IN THE BLUE BOXES AND THE
NUMBER OF -- THE MEDIAN DAYS FOR
TIME TO APPROVAL OF IRB
APPROVALS FOR THE PROJECTS IS
SHOWN IN THE ORANGE LINE.
YOU CAN SEE THE DRAMATIC
DECREASE CUT OVER BY HALF THE
TIME IT TAKES TO DO THE
APPROVALS.
THIS IS AN INCREDIBLE WAY TO
HELP MAKE THINGS GO FASTER AND
SO VERY EXCITING TO SEE THIS
FROM THE SMART IRB AND GET THIS
NATIONAL RECOGNITION OF THE WORK
THEY'VE BEEN DOING.
AND THEN OF COURSE ON A SAD
NOTE, MANY OF YOU KNOW THAT
THERE'VE BEEN MEMBERS THAT
PASSED AWAY.
DR. RAFFLE SACCO FROM UNIVERSITY
OF MIAMI, CTSA, HAS BEEN A
MEMBER OF OUR STEERING COMMITTEE
FOR THE FOR THE PROGRAM AND AND
VALUED MEMBER OF THE COMMUNITY
AND PASSED AWAY RECENTLY.
IN ADDITION, SUSAN SMITH FROM
THE UNIVERSITY OF ARKANSAS
MEDICAL SCHOOL IN TRI, THE
TRANSLATIONAL RESEARCH INSTITUTE
ALSO PASSED AWAY AND SERVED ON
THE STEERING COMMITTEE.
VERY VALUED MEMBERS OF THE CTSA
COMMUNITY.
LASTLY LARRY FROM THE UNIVERSITY
OF KENTUCKY, CCTS.
GERRY SUPINSKI AND SADDENED BY
THE NEWS OF OUR COLLEAGUES AND
FRIENDS ACROSS THE CTSA
CONSORTIUM.
EACH OF THEM WERE TOP-NOTCH
SCIENTISTS, SCHOLARS,
PHYSICIANS, MENTORS AND HUMANS.
SO THEY WILL BE DEEPLY MISSED
WITHIN OUR CTSA COMMUNITY AND
WE'LL CONTINUE TO DO GREAT
THINGS BECAUSE OF THE WORK THEY
HAVE ENABLED AS WELL.
ON ANOTHER NOTE SIMILARLY, LAST
TIME WE SPOKE ABOUT DR. BECKY
JACKSON WHO PASSED AWAY AS WELL.
SHE SERVED ON THIS COUNCIL.
WE ARE REALLY SAD TO SEE -- HEAR
THE NEWS BUT AT THE SAME TIME
AND THERE'S A REBECCA JACKSON
AWARD FOR ACHIEVEMENT IN
EDUCATION AND INNOVATION.
IF YOU'D LIKE TO LEARN MORE SEE
THE LINK SHOWN HERE.
THIS IS GRATIFYING TO SEE AND A
WONDERFUL WAY TO RECOGNIZE HER
CONTRIBUTIONS AND HER SPIRIT.
>>I WANT TO MOVE ON TO THE RARE
DISEASE COMMUNITY AND SPACE.
THERE'S A VARIETY OF ACTIVES
ONGOING IN THE SPACE ALL VERY
EXCITING AND I'M NOT ABLE TO
TOUCH ON ALL OF THEM AS WELL BUT
I WANTED TO REACH OUT AND TALK
ABOUT THE IDEA OF SHORTENING THE
DIAGNOSTIC ODYSSEY.
WE ALL KNOW 10% OF THE
POPULATION HAS A RARE DISEASE
BUT DON'T HAVE A VERY GOOD
ABILITY TO COUNT THEM IN OUR
ELECTRONIC HEALTH RECORD SYSTEM
OR OTHERWISE, FRANKLY.
THERE AREN'T VERY GOOD
DEFINITIONS.
IT MAKES IT HARD FOR PEOPLE TO
DIAGNOSE PEOPLE WITH RARE
DISEASES AND TO DEVELOP
THERAPIES.
THEY'RE LOOKING FOR WAYS TO
SHORTEN THE DIAGNOSTIC ODYSSEY
AND THINK OF HOW TO ENABLE
CLINICIANS OR THE PATIENT WHO'S
SEE THE CLINICIANS TO FIND WAYS
TO ENABLE A DIAGNOSIS FASTER.
I WANTED TO GIVE A SENSE OF WHAT
WE'RE DOING IN THE SPACE.
ONE OF THE PROGRAMS WE HAVE IS
ON SHORTENING THE DIAGNOSTIC
ODYSSEY FROM NCATS FROM THE
DRDRI PROGRAM AND HAVE THREE
ACTIVITIES IN THE SPACE.
ONE LOOKING AT HOW TO USE
ELECTRONIC HEALTH RECORDS TO
IDENTIFY RARE DISEASE PATIENTS
AND DEVELOP ALGORITHMS TO GO
BACK AND TEST AND SEE IF WE CAN
FIND ADDITIONAL RARE DISEASE
PATIENTS WITH THE SIMILAR
SYMPTOMS AND THEN VALIDATE THOSE
S ALGORITHMS AND SEE HOW THEY
WORK AND IMPORTANTLY IF YOU'VE
SEEN ONE ELECTRONIC HEALTH
RECORD IN ONE SYSTEM YOU'VE ONLY
SEEN ONE ELECTRONIC HEALTH
RECORD IN ONE SYSTEM.
AN IMPORTANT THING WE NEED TO DO
IS VALIDATING THIS IN A VARIETY
OF SYSTEMS TO MAKE SURE THOSE
ARLING
ALGORITHMS ARE ROBUST AND IN
ADDITION WE'RE LOOKING AT
A.I./M.L. APPROACHES TO IDENTIFY
DISORDERS AND IDENTIFYING THOSE
AND LASTLY TO THINK ABOUT
ENVIRONMENT PLATFORMS FOR
GENETIC EVALUATION IN UNDER
SERVED COMMUNITIES TO IDENTIFY
PEOPLE WHO HAVE RARE DISEASES
THAT AREN'T DIAGNOSED YET BUT
NEED SEQUENCING TO DO SO AND
THESE ARE AREAS WE'RE HOPING TO
EXPAND.
I'M HOPING IT'S A START IN
TACKLING THE DIAGNOSTIC ODYSSEY.
WE NEED TO DO MORE WORK.
I THINK THIS IS GOING TO BE
POWERFUL IN DECIDING WHAT NEXT
STEPS WILL BE IMPORTANT.
AND I'LL GIVE THE BRIEF OVERVIEW
WITH THE WITH KNOW OVER 80% ARE
SINGLE GENE DISORDERS AND MOST
ARE OFFSET IN EARLY CHILDHOOD.
IF WE KNOW THERE'S SINGLE GENE
DISORDERS IT GIVES A LEG UP ON
POSSIBLE APPROACHES FOR
TREATMENT.
THIS IS LED HERE AT NCATS AND
WE'VE DEVELOPED NOVEL
TECHNOLOGIES AND TESTED THEM IN
ANIMAL MODELS AND LOOKING FOR
SAFETY AND EFFICACY FOR MORE
APPLICATIONS NOW MOVING FORWARD.
THERE'S A NICE TOOL KIT FOR THE
DATA COMING FROM THE GENE
EDITING PROGRAM AND IF YOU'RE
INTERESTED I WANTED TO PUT THE
QR CODE FOR THAT AND IT'S
PUBLISH AND UNPUBLISHED DATA AND
YOU CAN TAKE A LOOK AT THAT.
FOR THE SECOND PHASE OF PROGRAM
WE'RE STILL DOING WORK ON
TECHNOLOGY DEVELOPMENT.
THAT'S ALWAYS IMPORTANT AND
WE'RE NOW TRANSITIONING INTO
PRE-CLINICAL AND IND STUDIES FOR
THE WORK UNDERSTANDING HOW TO
DEVELOP ASSAYS AND HOW TO MOVE
TOWARDS WHAT ARE ACTIVITIES
GOING TO BE NEEDED AND PERFORM
PERFORMING THOSE.
HOPEFULLY WE'LL MOVE INTO
CLINICAL TRIALS AS WE MOVE TO
THE LATER PARTS OF THE NEXT FIVE
YEARS OF THE PROGRAM.
STAY TUNED BUT I WANTED TO LET
YOU KNOW ABOUT THE NEXT PHASE
WE'RE MOVING INTO.
AND THE ACCELERATING MEDICINES
PROGRAM AND THE CONSORTIUM WITH
PJ BROOKS LEADING THE WAY AND QR
CODE IN THE BOTTOM.
THIS IS ABOUT DEVELOPING AND
EXPLORING THE ADENO ASSOCIATED
VECTOR AND THE BIOLOGY.
THERE'S SUB TYPES AND HOW TO WE
MAKE SURE WE'RE GETTING THE
RIGHT VECTOR TO THE RIGHT CELLS
AND DOSE.
THEY'RE ALL KEY QUESTIONS AND
OPTIMIZING THE VECTOR GENERATION
AND GENE EXPRESSION FOR THE
DELIVERY OF THOSE GENES AS
THERAPIES START TO ADVANCE.
AS WE LOOK AT THE RESEARCH AND
DEVELOPMENT PIECE, THERE'S A
REGULATORY SCIENCE PIECE HERE
TOO.
THE IDEA IS IF WE CAN VAEBS THE
VIRAL DEVELOPMENT OF THE VIRAL
CAPSIDS FOR GENE VIRAL DELIVERY
IS SOMETHING IN A REGULATORY
SENSE WE CAN TAKE OFF THE TABLE
BECAUSE THE SAFETY AND EFFICACY
WILL BE FAIRLY WELL KNOWN FOR
THE DELIVERY DEVICES.
THE REGULATORY ASPECTS NEED TO
FOCUS ON THE ACCESS OF THE
DELIVERY.
IT'S WAYS TO STREAMLINE THE
REGULATORY PROCESSES HERE AND
ALLOW WAYS TO MAKE IT AVAILABLE
FOR MORE RESEARCHERS.
THIS IS AN EXCITING PROGRAM AND
WE'RE NOW A YEAR INTO IT AND
HAVE A VARIETY OF PARTNERS FROM
INDUSTRY AND NON-PROFIT INDUSTRY
AND NIH WORKING ON THIS.
WE'RE TESTING THE APPROACHES AND
EXPECT TO HAVE FINAL SELECTION
BY MAY.
HOPEFULLY MAY COUNCIL I CAN GIVE
A DEEPER UPDATE ON THIS.
ONE OTHER IMPORTANT IN RARE
DISEASE IS THE NETWORK, RBCRN.
THIS EMBODIES OUR APPROACH OF
MANY DISEASES AT A TIME.
IT'S IMPORTANT WHEN WE THINK OF
OVER 10,000 RARE DISEASES.
WE CAN'T GO ONE BY ONE.
WE WANT TO THINK OF WAYS TO
LEVERAGE WORK BEING DONE ACROSS
DIFFERENT NETWORKS LOOKING AT
VERY SIMILAR SYMPTOMOLOGY.
THE RDCRN HAVE 20 GRANTS AND
LOOK AT LEAST THREE DIFFERENT
RELATED DISEASES.
THIS IS A WAY IN WHICH WE CAN
HONE IN ON THE MORE THAN ONE
DISEASE AT A TIME.
IN ADDITION, THIS IS ALSO A WAY
TO GO NATIONAL AND EVEN GLOBAL.
IMPORTANTLY, WITH RARE DISEASES
WE WANT TO TRY TO BRING IN AS
MANY PEOPLE WITH RARE DISEASES
AND RECOGNIZING THIS IS NATIONAL
AND INTERNATIONAL IS A KEY
ACTIVITY I WANTED TO HIGHLIGHT.
AND I'M ASSUMER PROUD AND HAPPY
TO SAY WE HAD AN OPPORTUNITY TO
DO A GUEST LOG AND LARRY TABAK
ASKED TO US DO A BLOG ON RARE
PUBLIC HEALTH CHALLENGE AND ALL
ABOUT RARE DISEASES AND SOME KEY
IDEAS I TALKED ABOUT ARE HERE.
LOOKING MORE IN DEPTH TO IDEAS
AND FINDING OUT WHAT WE'RE DOING
YOU CAN CLICK ON THE QR CODE AND
CHECK IT OUT.
THIS IS ONE OF THE CARTOONS
PUBLISHED FROM OUR RARE DISEASE
CHALLENGE THAT RARE DISEASES ARE
NOT RARE.
IT EMBODIES THERE'S MANY RARE
DISEASES AND ALL AMONG US.
OUR COMMUNITIES.
I THOUGHT I WAS A GREAT IMAGE TO
REACH OUT TO THE RARE
COMMUNITIES IN THAT WAY.
AND THIS IS RARE DISEASE DAY.
IT'S A DAY I CIRCLE EVERY YEAR
AND AN EXCITING MEETING AND
BRINGS TOGETHER THE VOICES,
SCIENTISTS, CLINICIANS, FUND ,
PATIENTS, PATIENT ADVOCATES AND
LEADERS WITHIN THE RARE DISEASE
COMMUNITY AND I'M SUPER EXCITED
TO HAVE THIS HAPPEN FEBRUARY 28.
AND THERE'LL BE AN OVERVIEW
ABOUT THAT PROGRAM AND I DON'T
KNOW HE'LL TALK ABOUT TISSUE
CHIPS SPECIFICALLY.
I WANTED TO HIGHLIGHT ONE THING
BECAUSE IT'S A MOMENT.
WE'VE DEVELOPED TISSUE CHIPS TO
GO UP INTO SPACE AND THEN TO DO
WORK HERE ON GRAVITY AS WELL.
SO WE HAVE GRAVITY AND
MICROGRAVITY EXPERIMENTS AT THE
SAME TIME AND WE'RE ABLE TO
ANALYZE THE DATA IN PARALLEL.
WE'VE HAD A VARIETY OF LUNCHES
IN THE LAST FEW YEARS AND THE
LAST LAUNCH WITH THIS PROGRAM IS
MARCH 2.
I WANTED TO MARK THE MOMENT
BECAUSE IT'S BEEN AN INNOVATIVE
AND EXCITING PROGRAM AND THANK
THE TEAM FOR THE INCREDIBLE WORK
AND THANK PARTNERS AT NASA FOR
THIS WORK.
I WANTED TO TAKE A MOMENT TO
RECOGNIZE THAT AND OUR
ASTRONAUTS FOR DOING SOME OF THE
WORK.
ARE NOW I'LL TURN TO ENDING ON A
FEW OF OUR NCATS COVID RESPONSE
UPDATES.
AS YOU KNOW, I HOPE WE'RE
ENTERING A PHASE WHERE IT'S
BEGINNING TO PHASE OUT.
IT'S A WILY VIRUS AND WE'RE
SEEING NEW PHASES AND WE'RE
MONITORING THIS THROUGH THE OPEN
DATA PORTAL I WON'T REALLY TALK
ABOUT TODAY BUT IT HELPS US KEEP
TRACK OF WHAT'S HAPPENING IN
TERMS OF THE EVOLUTION OF THE
VIRUS.
I WANTED TO MENTION WORK ON THE
NATIONAL COHORT COLLABORATIVE.
THIS IS A PROGRAM ON BRINGING IN
ELECTRONIC HEALTH RECORDS FROM
COVID AFFECTED INDIVIDUALS.
WE NOW HAVE OVER 17 MILLION
ELECTRONIC HEALTH RECORDS FROM
AND 17 MILLION TOTAL, 7 MILLION
ARE COVID POSITIVE.
AN INCREDIBLE RESOURCE FOR THIS
KIND OF DATA.
THERE'S BILLIONS ROWS OF DATA.
IT'S INCREDIBLE AND MORE AND
MORE PAPERS ARE COMING OUT AND
THERE'S AN EXCESS OF VARIETY OF
PAPERS ON SPECIFIC AREA OF LONG
COVID.
IT'S TURNING OUT THAT THEY FOUND
THE SEVERITY OF SARS COV2
REINFECTIONS WAS THAT TO THOSE
OF THE INITIAL INFECTION BUT THE
NUMBER OF LONG COVID DIAGNOSES
SHOWED REINFECTION WITH RECENT
VARIANTS.
IT'S A CASE IN POINT TO REMAIN
VIGILANT AROUND ACTIVITIES
RELATED TO THE VIRUS AND WHEN
IT'S RAMPANT IN OUR RESPECTIVE
AREAS.
TO DATE, THIS WORK HAS SHOWED A
LOT OF RESEARCH HIGHLIGHTING
WHAT'S GOING ON IN LONG COVID
INCLUDING THE PHENOTYPES THAT
ARE RELATED TO LONG COVID AS
WELL.
THERE'S BEEN 13 MANUSCRIPTS
SUBMITTED IN PROGRESS FROM THE
GROUP AND A VARIETY OF
COLLABORATIONS WITH THE RECOVER
PROGRAM AND ALL OF US RESEARCH
PROGRAM.
THIS HAS BEEN HIGHLIGHTED A
COUPLE TIMES IN THE NIH
DIRECTOR'S BLOG.
WE'RE GETTING GOOD ATTENTION IN
THE NIH DIRECTOR'S BLOG WHICH IS
GREAT IT SEE.
THIS IS AN ONGOING PROBLEM AND I
THINK THE CLINICAL DATA AND
ELECTRONIC HEALTH RECORD DATA
AND REAL-WORLD DATA AND EVIDENCE
ARE NEW TOOLS WE HAVEN'T HAD
BEFORE AND WE'RE LEARNING HOW TO
USE THE TOOLS AND IT'S TURNING
OUT TO BE QUITE POWERFUL.
I HOPE WE CAN CONTINUE TO DO
THIS KIND OF ACTIVITY.
ON THE ACTIVE SIDE ACCELERATING
COVID-19 INTERVENTIONS AND
VACCINES, I WANTED TO JUST
BRIEFLY UPDATE ON THE
PRIVATE-PUBLIC PARTNERSHIPS AND
THIS HAS BEEN A WONDERFUL
PARTNERSHIP.
I WANT TO HIGHLIGHT ACTIVITIES
WE'VE BEEN DOING.
I WON'T GO INTO TOO MUCH DETAIL
BUT OUR ROLE HAS BEEN FOCUSSED
ON STAGE 1 AND STAGE 2 SIDE.
THE OUT PATIENT POPULATION.
ACTIV FOCUSSED ON THE IN-PATIENT
POPULATION.
THERE WERE FINDINGS WHERE THEY
MISSED THE PRIMARY END POINT BUT
HAD GOOD SECONDARY END POINT IN
MORTALITY.
HOPEFULLY THERE'LL BE A
PUBLISHING SOON.
AND I WANT TO TALK ABOUT THE
OUT-PATIENT SETTING FOR THE
CLINICAL TRIALS.
IT'S ALL DECENTRALIZED.
WE SEND THE MEDICATIONS TO
PEOPLE'S HOME AND THEY RESPOND
BY TAKING THOSE MEDICATIONS AND
PROVIDING A SERIES OF
QUESTIONNAIRES, ANSWERS A
VARIETY OF QUESTIONS ABOUT
THOSE.
WHAT I WANTED TO BRIEFLY SHOW
HERE IS THAT THERE'S BEEN FOUR
ACTIVE AN NONE SHOWED BENEFIT
FOR COVID-19 AND WE LOOKED AT
IVERMECTIN AND FLUTICASONE AND
FLUVOXAMINE AND THE STUDIES ARE
STILL VERY IMPORTANT TO
UNDERSTAND AND WE HAVE SOME
MEDICATIONS SHOWING THIS IN THE
VACCINES AND ANTIVIRALS.
MONOCLONAL ANTIBODIES ARE
NOTHING AT THE MOMENT BUT
PERHAPS THOSE WILL COME BACK ON
THE SCENE EVENTUALLY.
I THINK THAT EVEN THOUGH THESE
ARE NOT SHOWING BENEFIT THEY'RE
STILL VERY IMPORTANT TO REALLY
RELATE TO THE SCIENTIFIC
COMMUNITY AND TO THE CLINICIANS
TREATING PATIENTS WITH COVID-19.
AND ONE MORE ANECDOTE ON ACTIVE
6, I WANTED TO HIGHLIGHT IS THAT
THIS DECENTRALIZED APPROACH HAS
BEEN EXTRAORDINARY POWERFUL IN
REDUCING THE TIME IT TAKES TO
MEET ENROLLMENT TARGETS AND ALSO
GO FROM STUDY ACTIVATION TO
RELEASING THE PRE PRINT AND THAT
IS REALLY INCREDIBLE TO SEE AND
AGAIN I THINK IT SHOWS
ROBUSTNESS OF THE PROGRAM, THE
TEAM ON ACTIVE 6 HAS BEEN
INCREDIBLE OUT OF DCRI AND A
VARIETY OF GROUPS INVOLVED IN
RECRUITING AND ENROLLING THE
PATIENTS.
THIS IS A BIG NETWORK AND IT'S
BEEN VERY POSITIVE.
THE ONE THING I WANTED TO POINT
OUT TOO ON THIS IS THAT THESE
PEER-REVIEWED ARTICLES THOUGH
THEY'RE NOT SHOWING BENEFIT IN
TERMS OF COVID-19 I THINK THE
COMMUNITY SEIZE -- SEES BENEFIT
AND IN JAMA THIS IS THE NUMBER
ONE ARTICLE IN TERMS OF OUTPUT
FOR JAMA.
IT'S FASCINATING TO SEE.
AND FORBES MAGAZINE QUOTED THIS
STUDY AND THEY SAID THIS TYPE OF
STUDY PROVIDE THE MOST ACCURATE
AND UNBIASSED RESULTS AS NEITHER
THE PARTICIPANTS NOR THE STUDY
INVESTIGATORS KNOW WHETHER THE
PARTICIPANTS RECEIVE PLACEBO OR
DRUGS AND NEITHER CHOSE WHICH
ONE TO GET.
I THOUGHT THIS WAS A VERY -- A
GOOD REMINDER THESE ARE BLINDED
STUDIES, RANDOMIZED PLACEBO
CONTROLLED TRIALS AND THE
VALIDITY OF THE RESULTS.
I WANTED TO HIGHLIGHT THAT AND
CONGRATULATE THEM FOR THE WORK
THEY'VE DONE IN THE SPACE.
THAT WAS A WHIRLWIND TOUR OF
SOME HIGHLIGHTS HAPPENING SINCE
WE LAST MET.
AND SO JUST TO SUMMARIZE AGAIN
HERE I THINK RECOGNIZING THE
UNUSUAL I THINK LOSS WE'VE HAD
OF INDIVIDUALS WITHIN OUR
COMMUNITIES WITH HEAVY HEARTS
FOR THEM BOTH PERSONALLY AND
PROFESSIONALLY.
I WANTED TO TAKE A MOMENT TO
RECOGNIZE THAT.
IT'S ABOUT RELATIONSHIPS AND
ABOUT PEOPLE.
THIS IS WHAT WE DO.
SO IT'S REALLY IMPORTANT TO TAKE
A MOMENT TO RECOGNIZE THAT IN
OUR COMMUNITY.
AT THE SAME TIME WE'RE TO STAND
FIRM ON OUR FOUNDATION.
HAPPY TO BE IN THE PERMANENT
ROLE TO LEAD US INTO THE NEXT
DECADE, MOVE FORWARD IN WAYS I'M
GOING TO WORK WITH THE COMMUNITY
ON AND GET BUY-IN AND INPUT AS
WE CO-DESIGN AND CO-DEVELOP WITH
THE STRATEGIC PLAN LOOKS LIKE TO
BE MOST EFFECTIVE AS POSSIBLE.
I SHARED A VARIETY OF IMPACTFUL
ADVANCES AND EXCITED ABOUT
ENGAGEMENT WITH OUR STAKEHOLDERS
SUCH AS THE CTSA ROUNDTABLES.
I THINK IT WILL CONTINUE TO LEAD
TO MORE ADVANCEMENTS I'M EXCITED
TO SEE.
I LOOK FORWARD TO CARRYING OUT
THE DISCUSSIONS FORWARD TO
CREATE OUR NEXT ITERATION OF THE
NCATS STRATEGIC PLAN.
ONWARD WE GO AND THANK YOU FOR
YOUR ATTENTION AND GUIDANCE AND
INPUT AND LOOK FORWARD TO ANY
QUESTIONS OR DISCUSSIONS YOU
MIGHT HAVE NOW.
>>THANK YOU.
THIS WAS AN AMAZING AMOUNT OF
INFORMATION AND WE'RE GRATEFUL
FOR THE WORK YOU DO AND PACE
WITH WHICH YOU DO IT SO THANK
YOU.
I HAVE A QUESTION ABOUT THE
STRATEGIC PLANNING EFFORT AND
OPPORTUNITIES FOR COMMUNITY
ENGAGEMENT.
I'M SURE I KNOW A LOT OF PEOPLE
ATTEND THE COUNCIL MEETINGS AND
HAD A MEETING WITH PARTNERS
YESTERDAY WHO ARE INCREASING
PLAY PAYING ATTENTION TO THE
ROLE NCATS WITHIN THE RARE
DISEASE COMMUNITY.
I'M WONDERING IF YOU CAN TALK
ABOUT YOUR PLAN FOR ENGAGEMENT
TO THE COMMUNITY AND TALK ABOUT
WHAT THE PAST STRATEGIC PLAN WAS
AND HOW MILESTONES AND TIME
LINES WERE ACHIEVED AS YOU WORK
TO ENGAGE FOLKS AS YOU WANT
PEOPLE TO BE EDUCATED ON WHAT
WAS ACHIEVED AND WHAT THE
HURDLES WERE AND HOW PEOPLE CAN
COME TO THE TABLE READY TO
INFORM THAT PLAN GOING FORWARD.
>>I'LL TACKLE THE FIRST PART
FIRST.
IT'S A GREAT QUESTION.
WE'RE IN THE MIDST OF DEVELOPING
WHAT THEY'LL LOOK LIKE AND
THERE'LL BE A SERIES OF IN
PERSONAL AND EXTERNAL ENGAGEMENT
BECAUSE IT'S IMPORTANT TO MAKE
SURE WE TALK WITH OUR INTERNAL
TEAMS AND THE WE'RE TRYING TO
FIGURE OUT HOW TO BEST MAP THOSE
OUT.
ONE IS TO THINK ABOUT HOW TO
LOOK AT THE STRATEGIC PLAN AND
HIGHLIGHT DISCUSSIONS AROUND
THOSE.
WE'LL PROBABLY DO A MIXTURE OF
ALL OF THESE.
IT WILL PROBABLY A YES AND
ANOTHER OPTION ABOUT ENGAGING
COMMUNI
COMMUNITIES I'D LIKE IT REACH
OUT TO STAKEHOLDER AND
COMMUNICATE IF THERE'S
CONVENINGS THEY'RE HOLDING WHERE
THERE MAY BE AN OPPORTUNITY TO
TALK WITH COMMUNITIES AND GET
ADDITIONAL INPUT IN THAT WAY
THAT WOULD BE VALUABLE.
A
AND DOESN'T BURDEN US TO FIND A
DAY EVERYBODY CAN DO AND RATHER
COME TO YOU.
THAT'S PART OF THAT PLAN.
A THIRD PART OF THAT PLAN IS
ONCE WE DRAFT UP SOME OF THE
PLANS WE'D DO A REQUEST FOR
INFORMATION FOR ANYONE THROUGH
THE TYPICAL CHANNELS AT NIH.
THAT'S A WAY TO GET BROAD IMPACT
AND FEEDBACK ON THE SPECIFICS
AROUND THE PLANS AND GET INPUT
FROM THE COMMUNITY.
THAT'S A THIRD WAY WE CAN DO IT.
I THINK IT'S A HIGHER LIFT FOR
MOST TO DO THAT.
IT'S IMPORTANT TO ME TO AND GET
PLANS ON WHERE THEY CAN ADD
VALUE.
IT'S IMPORTANT TO DO THE
LISTENING AND UNDERSTAND WE'RE
ALL PART OF THE ECO SYSTEM AND
WHERE CAN WE PLAY WITH EACH
OTHER'S STRENGTHS AND CAPITALIZE
ON WORK ONGOING AND NOT
DUPLICATE EFFORTS BUT EXPAND ON
THEM AND COMPLIMENT THEM.
MOST IMPORTANTLY, ENABLE THE
IDEAS AND SO HEARING FROM THE
COMMUNITY HELPS US KNOW WE'RE ON
THE RIGHT TRACK OR MAYBE NEED TO
SHIFT GEARS TO SOMETHING
DIFFERENT THAT CAN HELP OTHER
AREAS.
THOSE ARE THE KINDS OF THINGS
WE'LL BE LOOKING AT.
BY NO MEANS IS THIS SORT OF
CLOSE TO BEING FULLY
ARTICULATED.
WE HAVE GOALS WE'D LIKE TO TALK
ABOUT AND THINK BY TALKING ABOUT
THE SPECIFIC GOALS, YOU
MENTIONED METRICS AND HOW DO WE
KNOW WE'RE ADVANCING ON THOSE?
WE WANT TO HIGHLIGHT AREAS TO
GET CONCRETE ABOUT THAT.
AND IF WE DO FAIL I WANT TO FAIL
FORWARD SO ULTIMATELY WE CAN GET
THERE.
WE CAN ULTIMATELY GET THERE.
I DON'T WANT TO BE AFRAID OF
FAILURE.
I WANT TO PUSH THE ENVELOPE AND
WORKING WITH THE STAKEHOLDERS
AND COMMUNITY WILL BE CRITICAL.
>>I THE THIS NOT HAPPENING WITH
NCATS BUT WITH OTHER GROUPS IS
DUPLICATING EFFORT AND THERE
SHOULD BE A FAIR AMOUNT OF
CONVENING AND WITH COLLEAGUES
AND RESEARCH CONSORTIUM WHICH I
KNOW NIH INTERACTS WITH BUT HAS
TIE-INS.
THERE'S SO MANY THINGS AROUND
THE GLOBE RIGHT NOW I DON'T WANT
US TO DO ANY ME TOOs IF WE CAN
AVOID IT BECAUSE THERE'S WORK TO
BE DONE BUT LOVE THE SUMMARIES.
AVOID IT BECAUSE THERE'S WORK TO
BE DONE BUT LOVE THE SUMMARIES.
AVOID IT BECAUSE THERE'S WORK TO
BE DONE BUT LOVE THE SUMMARIES.
RARE DISEASE IS A GREAT PLACE
FOR NCATS TO BE.
>>I COULDN'T AGREE MORE.
A LOT OF WHAT WE DO -- SOME
PEOPLE ARE CONCERNED SOMETIMES
WE MAY DUPLICATE EFFORT NOT
BECAUSE WE'RE ACTUALLY
DUPLICATING EFFORT BUT TALK
ABOUT SPECIFIC DISEASES
SOMETIMES TO MAKE THE POINT THAT
WE LOOK AT RARE DISEASES AS A
WHOLE.
YOU CAN'T DO TRANSLATIONAL
SCIENCE WITHOUT DOING
TRANSLATIONAL RESEARCH.
UNDERSTANDING HOW TO DO RESEARCH
ON SPECIFIC DISEASES TRYING TO
UNDERSTAND HOW TO DO THAT FOR
MORE THAN ONE DISEASE AT A TIME
HELPS US GET THERE AND TALK
ABOUT AREAS AND SAY SOME
DUPLICATION IS GOOD BECAUSE
THERE'S VALIDATION WITH THAT AND
WE WANT TO BE SMART ABOUT IT AND
WE'RE IN A GOOD POSITION TO DO
THAT AND YOU MAY KNOW PJ BROOKS
IS SERVING ON THE INTERNATIONAL
COMMUNITY WE HAVE GOOD PRESENCE
THERE AND WE'RE PAYING ATTENTION
TO.
AS WE MOVE FORWARD THESE ARE
THINGS WE WANT TO KEEP AT THE
FOR FRONT TO ENABLE THE WORK WE
DO TO DO WHAT YOU'RE TALKING
ABOUT.
SURGE YO.
-- SERGIO.
>>THANK YOU.
IT'S REMARKABLE THE PROGRESS
MADE.
YOU PIQUED MY INTEREST IN
ENGAGING WITH THE COMMUNITY AND
LISTENING TO THE COMMUNITIES.
AND WE HAVE BEEN MOVING IN THE
SPACE OF WHICH COMMUNITIES ARE
YOU LISTENING TO AND EVERS ON
HEALTH EQUITY AS WELL.
I WANT TO REACH OUT TO THE
COMMUNITIES THAT CAN BENEFIT
FROM THE WORK WE DO AND HAVEN'T
BENEFITTED AS WELL.
>>I KNOW I'LL BE ABLE TO LEARN
FROM YOU IN THE SPACE TOO.
THERE'S BEEN VARIETIES FROM THE
CTSA PROGRAM TALKING ABOUT IDEAS
OF DIVERSITY, EQUITY, INCLUSION
AND ACCESS AND IT'S ABOUT
LISTENING AND ENGAGING
COMMUNITIES.
DO WE NEED TO CONSIDER BEST
PRACTICES OF ENSURING WE DO THAT
NOT JUST DIVERSITY, EQUITY,
INCLUSION AND ACCESSIBILITY BUT
REACHING OUT TO THEM AND PERHAPS
BRINGING THEM INTO OUR STAFF FOR
DOING CLINICAL TRIALS.
THINGS OF THAT NATURE.
THIS IS WHY WE NEED THE DIALOGUE
AND ENGAGEMENT AND LISTENING.
AND IN TERMS OF THE STRATEGY
PLANNING PROCESS AND THE
ENGAGEMENT AND THE ROUNDTABLE
DISCUSSIONS I MENTIONED, WE'D
HOPED TO EXPAND -- WE DON'T PLAN
TO HOPE, WE'LL BE EXPANDING IN
PROBABLY IN MARCH.
WE'RE JUGGLING A LOT OF THESE
HEAVY CALENDAR LOADS HERE BUT
IT'S IMPORTANT BECAUSE THIS IS
THE TIME TO REALLY SLOW DOWN AND
MAKE SURE WE'RE LISTENING AND
TOUCHING BASE TO BE ON THE RIGHT
TRACK SO WHEN WE HAVE GONE
THROUGH THE OTHER SIDE OF THE
PROCESS WE CAN SPEED UP AND HAVE
MORE TRUST I HOPE WITH THE
COMMUNITY TO MOVE FORWARD
TOGETHER AND CONTINUE TO BUILD
THOSE TIES.
THE CTSA ROUNDTABLE STARTED WITH
THE PRINCIPLE INVESTIGATOR
LEADERSHIP AND INSTITUTIONAL
LEADERSHIP BUT AS WE GO FORWARD
IN THE NEXT ROUNDS OF THE
ROUNDTABLE DISCUSSIONS WE'LL BE
REACHING OUT MORE TO OTHER
ASPECTS OF THE CTSA AND A CHANCE
TO TALK TO THE ADMINISTRATORS
AND THEY'LL BE ON THE LIST AND
TRAINING DIRECTORS WILL BE ON
THE LIST AND COMMUNITY
ENGAGEMENT FOLKS WILL BE ON THE
LIST.
THAT'S ONE WAY IN WHICH WE CAN
DO THAT.
I'D BE HAPPY IF YOU HAVE ANY
OTHER THOUGHTS ON THIS SERGIO,
ALWAYS HAPPY TO HEAR HOW WE CAN
EVEN DO MORE AND EXPAND THEM.
>>ARE YOU THINKING ABOUT
LISTENING SESSION WITH MEMBERS
FROM MAYBE THE CTSA -- MOST HAVE
A COMMUNITY ADVISORY BOARDS THAT
ARE GOOD EXTENSION TO THE
PUBLIC.
>>GREAT QUESTION.
I DON'T QUITE KNOW YET HOW TO DO
THAT AND WOULD LOVE TO FIGURE
OUT HOW TO ACCESS THAT IN A WAY
THAT'S UNIFIED OR DO A
ROUNDTABLE WE JUST INVITE THE
CABS TO THE TABLE ON A COUPLE
DATES WE CAN PICK TO ALLOW IF
THE CAN'T MAKE ONE DAY PERHAPS
ANOTHER.
UNDERSTANDING HOW TO PIGGYBACK
ON EXISTING MEETINGS IS ONE WAY
AND ALSO THINKING ABOUT WHAT'S
THE COMPLETE LIST WE WANT TO
MAKE SURE WE REACH OUT TO THAT
MAY NOT HAVE SPECIFIC CONVENINGS
BUT WANT TO TAP INTO.
GREAT QUESTION PAUL AND IF YOU
HAVE THOUGHTS HOW BEST TO DO
THAT, I'D LOVE TO HEAR IT AND
MAYBE WE CAN TALK FURTHER ON
THAT.
AND PROBABLY IN MAY WE'LL GIVE
UPDATES AND WOULD LOVE YOUR
PARTICIPATION IN THOSE WHEN THEY
DO COME YOUR WAY.
IF THERE'S NO OTHER QUESTIONS,
ANNA, HOW WILL WE DOING ON TIME.
>>WE'RE SLIGHTLY AHEAD OF
SCHEDULE SO IF CHRISTINE IS
READY TO GO, WE CAN MOVE HER
CONCEPT CLEARANCE UP BY A LITTLE
BIT.
>>I'M HERE AND READY TO GO.
>>ALL RIGHT.
>>I'LL GIVE A BRIEF
INTRODUCTION BEFORE I INTRODUCE
THE OFFICE I WANTED TO INTRODUCE
A NEW STAFF MEMBER IN THE
PARTNERSHIP PROGRAM SHE WENT ON
TO DO A POST DOCK IN HARVARD IN
D
GENETICS AND COMPLEX DISEASE.
SHE SPENT TIME AT REGENERON
MANAGING A TEAM ON TARGET
DISCOVERY AND PHARMACOLOGY FOR
OBESITY AND PRE-DIABETES AND HAD
BEEN A DIRECTOR FOR A KOREAN
COMPANY FOR PLANNING FOR NEW
BUSINESS DEVELOPMENT BEFORE SHE
JOINED US IN THE OFFICE OF
DIRECT DEVELOPMENT PARTNERSHIP
PROGRAMS AT NCATS.
>>AND I LOOK FORWARD TO WORKING
WITH YOU.
SO OKAY, LET'S GO ON TO THE
FIRST SLIDE FOR INTRODUCTION
HERE.
WE DEVELOP PARTNERSHIPS THAT
PROMOTE INNOVATION AND FOCUSSED
ON FILLING GAPS IN PRE-CLINICAL
TRANSLATIONAL SCIENCE FROM EARLY
DISCOVERY THROUGH EARLY STAGE
CLINICAL TRIALS.
AND REALLY HAVE AN EYE TOWARD
DEVELOPING TECHNOLOGIES AND
STRATEGY ARE GOING TO IMPROVE
DIVERSITY, EQUITY INCLUSION AND
ACCESS IN HEALTH CARE.
ON THE NEXT SLIDE THESE ARE THE
ONGOING PROGRAMS WE HAVE RUNNING
NOW IN THE OFFICE AND THINGS IN
GRAY ARE WHERE FINANCIAL SUPPORT
IS COMING FROM OFFICE OF THE
DIRECTORS AND DIRECTING THE
GENOME PROGRAM AND IT'S AN NIH
COMMON FUND PROGRAM AND ENDING
ADDICTION LONG-TERM IS THE LARGE
INITIATIVE IN RESPONSE TO THE
OPIOID CRISIS.
EVERYTHING LISTED IN PURPLE ARE
INITIATED AND GENERALLY FUNDED
BY NCATS.
NOW ON THE NEXT SLIDE WE WILL
GET INTO THE ACTUAL CONCEPT
WE'RE CALLING PRE CLINICAL
CONCEPT STUDIES FOR RARE
DISEASES.
LOOKING AT THE SAME DRUG
DEVELOPMENT PIPELINE, WE WANTED
TO BREAK DOWN A LITTLE BIT
FURTHER FOR YOU THE PRECLINICAL
DEVELOPMENT CHEVRON INTO TWO
STAGES.
ONE BEING PRECLINICAL PROOF OF
CONCEPT AND THE IND-ENABLING
DEVELOPMENT AND THE
DEMONSTRATION OF EFFICACY IN A
MODEL SYSTEM BEFORE YOU WOULD
MAKE A LARGE INVESTMENT FOR
DOING THE REGULATORY SAFETY
STUDIES YOU'RE GOING TO NEED
BEFORE YOU WOULD ACTUALLY GO
INTO A HUMAN STUDY AND CLINICAL
TRIAL.
THE NEXT SLIDE ILLUSTRATES THE
GAP THAT WE'RE TRYING TO
ADDRESS.
SO PARTICULARLY WHEN WE SPOKE
WITH OUR INTRAMURAL COLLEAGUES
AND STEVE PITTINGER THAT DOES A
LOT OF WORK WITH THEM NOTICED
THERE WAS A GAP BETWEEN PERHAPS
IN THE EARLY DISCOVERY AND EARLY
PRE-CLINICAL DEVELOPMENT WHERE A
LOT OF TIMES AN INVESTIGATOR OR
COLLABORATOR MAY BE FOCUSSED ON
DEVELOP A PROBE TO DO FURTHER
RESEARCH AND THEN IN THE TEAL
COLOR WE HAVE THE STUDIES BY THE
THERAPEUTICS DEVELOPMENT BRANCH.
THIS AND THIS TENDS TO BE NO
MATTER WHERE YOU ARE AND SEE IT
IN THE EXTRAMURAL PROGRAM BUT I
THINK IF YOU WENT THROUGH
ACADEMIC INSTITUTIONS YOU'D SEE
THE SAME AND THE GROUPS THAT DO
THE ENABLING WORK IS A DIFFERENT
SKILL SET AND FOCUS.
WHEN YOU GET SOMEBODY TO THE
POINT WHERE THEY HAVE A PROBE
THAT COULD CONTINUE ON FOR
DEVELOPMENT YOU NEED TO STUDY
THIS IN SOME MODEL SYSTEM AND
THIS COULD BE QUITE COSTLY.
WE WANTED TO HAVE A FUNDING
OPPORTUNITY THAT WOULD HELP TO
ADDRESS THIS.
THE GOAL OF THE OPPORTUNITY IS
TO BRIDGE THE GAP BETWEEN THE
DEVELOPMENT AND IND ENABLING
STUDIES WHEN HAVE YOU A
MERITORIOUS COMPOUND AND WE'RE
FOCUSED ON RARE DISEASES AND
THAT'S THE MOST EFFECTIVE PATH
FOR GETTING TREATMENT TO PATIENT
SO WE'D ALSO WANT TO SUPPORT
THAT AND OFTEN WANT TO SEE
EFFICACY IN MODELS BEFORE WE GO
INTO CLINICAL TRIAL.
THE CONCEPTS FOCUSES ON THE
IDEA.
IT'S SIMPLE.
THIS IS TO USE AN ESTABLISHED
RARE DISEASE MODEL WHETHER
ANIMAL MODEL OR TISSUE CHIP OR
SOME OTHER TYPE OF MODEL TO DO
THAT TESTING AND DEMONSTRATE THE
ADVOCACY.
ON THE NEXT SLIDE WE WANT TO
DEVELOP AN OPPORTUNITY THAT
FILLS THE GAP FOR RARE DISEASE
DRUG DEVELOPMENT BY SPECIFICALLY
PROVIDING FUNDING TO TEST
EFFICACY IN MERITORIOUS
COMPOUNDS IN AN ESTABLISHED RARE
DISEASE MODEL.
IN ADDITION WE WANT A STEERING
COMMITTEE.
WE WANT TO STRENGTHEN POTENTIAL
APPLICATIONS BY BRINGING THAT
PATIENT PERSPECTIVE INTO THE
PROCESS EARLIER THAN PEOPLE ARE
TYPICALLY THINKING ABOUT IT AND
MAY THINK ABOUT WHEN DESIGNING
THE CLINICAL TRIAL.
A LOT OF TIMES THEY'RE NOT
THINKING ABOUT IT AT THE EARLIER
STAGES.
WE'RE HOPING IN DOING THIS AND I
MENTION THE EARLY DISCOVERY
RESEARCHERS THOSE GENERALLY
FOCUSSED ON DEVELOP IING A PROB
WORKING ADJACENT TO DRUG
DEVELOPMENT AND MAYBE IT'S NOT
REALLY ON THEIR RADAR THAT THERE
IS THIS POTENTIAL TO KEEP GOING
FORWARD AND HAVE THE POTENTIAL
TO TRANSLATE WHAT THEY'RE DOING
INTO SOMETHING THAT WOULD HAVE
AN IMPACT IN THE CLINIC.
WE HOPE BY HAVING THE
OPPORTUNITY LIKE THIS IT WILL
MAKE SOME RESEARCHERS THINK
ABOUT THE POTENTIAL AND WORK
MORE INTENTIONALLY TOWARDS
TRANSLATING THE WORK THEY'RE
DOING AND THE COMPOUND THEY'RE
DEVELOPING.
AND WE THINK THIS RARE DISEASE
IS A VERY NCATS THING AND
ENGAGING THEM EARLY ON IN THE
DEVELOPMENT PROCESS.
IT FITS WELL WITH THE WAY NCATS
LIKES TO OPERATE AND INCREASING
THE USE OF EXISTING RARE DISEASE
MODELS ON WHICH THE MERITORIOUS
PROBES AND THERAPEUTICS WILL BE
TESTED AND TO THINK MORE
INTENTIONALLY IN DESIGNING
STUDIES TO WITH STAND CRITICAL
EVALUATION BY INDUSTRY AND HAVE
MORE RELEVANCE FOR PATIENT
POPULATIONS IN QUESTION WITH
THIS WE MAY GET MORE BASIC
SCIENTISTS THINKING HOW THEIR
WORK WILL IMPACT PATIENT LIVES.
WE WANT TO PROVIDE STRONG
PRE-CLINICAL EFFICACY AREA TO
TRACT INVESTMENT IN THE
DEVELOPMENT IN THE FIELD OF RARE
DISEASES.
FOR IMPACT, THE PENSION PROCEEDS
TO DEVELOPMENT ON OTHER PROGRAM
TO ENTER CLINICAL TRIALS IS OF
COURSE A SIGNIFICANT IMPACT WE
COULD HAVE HERE.
THE LAST SLIDE BEFORE I GET TO
QUESTIONS AND ENGAGEMENT WITH
YOU, IN TERMS OF THE CRITERIA
FOR EVALUATING SUCCESS, I THINK
THERE'S SEVERAL DIFFERENT WAYS
TO EVALUATE SUCCESS BUT THE
MAJOR ONES ARE LISTED HERE TO
EVALUATE THE NUMBER OF COMPOUNDS
THAT TRANSITION INTO FULL IND
ENABLING PRECLINICAL
DEVELOPMENT.
AT THE END OF THE DAY THAT'S
WHAT WE'RE TRYING TO DO HERE.
ALSO THE TRADITIONALLY NOT
TRANSLATIONAL SCIENTISTS WE
START TO PULL INTO THE SPACE I
THINK WOULD BE ANOTHER MEASURE
OF SUCCESS FOR THE PROGRAM.
SO EVEN AT THAT EARLY PHASE OF
THE PROGRAM WE WOULD BE ABLE TO
SEE AN EARLY INDICATOR WHETHER
OR NOT THAT'S REALLY HAPPENING.
AND THEN SOME OF THE OBSTACLES
OR BARRIERS OR THINGS WE MIGHT
ANTICIPATE WOULD BE THE NEED TO
ENSURE DRUG CANDIDATES THAT ARE
BEING PROPOSED AND REVIEWING THE
APPLICATIONS.
WE ALSO WANT TO ENSURE THE BEST
RARE DISEASE MODELS ARE BEING
UTILIZED.
AND WE WANT TO ENSURE THE RARE
DISEASE COMMUNITY IS AWARE OF
THE OPPORTUNITY SO THEY CAN
ASSIST WITH THE DEVELOPMENT OF
THE PARTNERSHIPS.
IN TERMS OF ONGOING RESEARCH AND
OPPORTUNITIES IN THE AREA WE'VE
WORKED TO ENSURE WHAT WE ARE
DOING IS GOING TO BE
COMPLIMENTARY TO AND NOT
COMPETING WITH OR OVERLAPPING
WITH WHAT THEY'RE DOING.
THIS IS PARTICULARLY NICE IN
THAT IT IS UP STREAM OF THE
CLINICAL TRIAL READINESS THEY
HAVE.
PARTICULARLY WHEN WE TALK ABOUT
REPURPOSING A MARKETED
THERAPEUTIC THIS COULD HELP FEED
THAT PIPELINE.
AND A LOT OF THIS WAS INITIATED
WITH ABSORBING AND SEEING WHAT
IS HAPPENING IN OUR OWN
INTRAMURAL RESEARCH PROGRAM AND
RECOGNIZING THE GAP EXISTS
BEYOND NCATS AND WANTING TO HELP
ADDRESS THAT.
IT'S IT FOR THE SLIDE AND IN THE
NEXT SLIDE I'LL GO OVER
QUESTIONS FOR YOU.
WE'RE INTERESTED TO HEAR IDEAS
WHAT MIGHT IMPROVE THE
INITIATIVE WHAT ARE KEY POINTS
WE SHOULD BE CONSIDERING AND
SPECIFICALLY VERY INTERESTED TO
HEAR IF YOU HAVE IDEAS HOW TO
FACILITATE DEVELOPMENT OF THE
PARTNERSHIPS WITH THE RARE
DISEASE COMMUNITY AND WHAT ARE
THE BEST METRICS USED TO
EVALUATE THE ASSET AND EVALUATE
THE RARE DISEASE MODEL.
OF COURSE ANY ADDITIONAL
COMMENTS YOU HAVE ARE ALSO
HELPFUL.
>>I THINK I'M ONE OF YOUR
REVIEWERS FOR THIS ONE.
I ENJOYED LOOKING THROUGH IT.
I THINK IT'S A GREAT PROGRAM.
A COUPLE SMALL COMMENTS.
ONE, I'VE SEEN A TREND AND I
SUSPECT OTHERS WHERE WE'VE SEEN
FAMILY ORGANIZATIONS AND PATIENT
ORGANIZATIONS GOING OUT AND
DEVELOPING ANIMAL MODELS OR
BIOLOGICAL MODELS FOR RARE
DISEASE.
OUR MIND SET HAS BEEN
RESEARCHERS DEVELOP THEM AND
THEY HAVE TO FIND THE FAMILIES.
WE'RE SEEING THE OPPOSITE AND
JUST IF YOU CAN FIND A WAY TO
SWAP THAT INTO YOUR MATCHING
PROCESS.
SO SOMETIMES WE WENT OUT AND
PAID SOMEBODY TO BUILD THE MOUSE
MODEL.
LET'S PAIR THEM UP WITH PEOPLE
DOING THE BASIC BIOLOGY OR
THERAPEUTIC INTERVENTION TRIALS,
THINGS LIKE THAT.
THAT'S A VERY NCATSIAN THING SO
AND I LOVE THAT PHRASE SO THANK
YOU FOR THAT.
>>ARE YOU SAYING IN SOME WAY
PARTNER WITH THOSE FAMILIES --
>>A WAY TO REFERRING RESOURCES.
YOU'D BE AMAZED HOW MANY GROUPS
WE GET REACHED OUT TO BY.
MAYBE TWICE A MONTH AND IT'S WE
PAID SO AND SO TO DEVELOP THE
MODEL.
BUT THEN THEY DON'T REALLY KNOW
WHERE TO GO NEXT WITH IT.
>>THAT'S GREAT.
>>LIKE I SAID, WE HAVE OUR
MODEL AND THE PATIENTS TRYING TO
HELP FUND THE RESEARCH AND NEED
TO GO FIND INVESTIGATORS.
THE OTHER IS PROBABLY OUT OF
NIH'S CLASSIVE COMFORT ZONE, I'D
ADVERTISE THE HELL OUT OF THIS.
THIS NEEDS TO BE IN FRONT OF OR
GLOBAL WE WANT TO PUSH THIS
FORWARD AND CAN BE A GREAT
LEAD-IN PROJECT FOR FAMILY
ORGANIZATIONS OR THE OTHERS.
IT'S ONE WHERE I'D GET OUT AND
PUSH AS A CAPTURE FOR WHAT'S
GOING ON.
THAT'S HOW TO BE SUCCESSFUL IF
YOU'RE GOING TO TRY TO MATCH
THESE THINGS UP.
>>RIGHT YEP.
TH
THAT'S GREAT.
>>I LOVE THE PROGRAM.
>>THANK YOU.
>>SURE THING.
>>THIS IS TED HOLMAN I THINK
I'M ALSO ON THE REVIEWER LIST.
NICE PROGRAM.
NOT SURE I HAVE SPECIFIC -- I'LL
TELL YOU FROM MY PERSONAL
EXPERIENCE.
WE DEVELOPED A LOT OF SMALL
MOLECULES IN MY LAB AND NORMALLY
WE TARGET LARGE DISEASES AND WE
HAVEN'T GOTTEN FUNDING IN THE
LARGE DISEASE PROJECTS AND WE'VE
HAD TO PIVOT AND ORPHAN DISEASE
DESIGNATION HELPS WITH FINDING
INVESTORS.
THERE'S A LOT OF INVESTMENT
STRATEGIES THAT PUSH YOU TO GO
ORPHAN FIRST, GET YOUR FDA
APPROVAL WITH ORPHAN INDICATION
AND PIVOT TO THE LARGER DISEASE
REQUIRE MORE FUNDING FOR
CLINICAL TRIALS, ETCETERA.
ONE HUGE CHALLENGE AS AN
ACADEMIC IS TRYING TO FIND THE
ORPHAN INDICATION THAT CAN
POTENTIALLY BENEFIT FROM MY
DISCOVERY.
WE'VE HAD TO DO A LOT OF LEG
WORK AND FIGURE OUT WHAT'S GOING
ON.
I DON'T KNOW HOW NCATS CAN DO
THIS BUT I'M SURE YOU GUYS HAVE
A LOT OF DATA MINING EXPERTS
WHERE YOU SAID IF YOU HAVE THE
TARGET MY POTENTIAL ONE IS
OXYGENASE AND IT'S IMPLICATED IN
A LOT OF DISEASES BUT HELP THEM
A, FIND THE ORPHAN INVITATION
THAT COULD BE BENEFICIAL TO THE
SMALL MOLECULE.
YOU HAVE PATHWAYS THAT
IMPLICATED IN MANY ORPHAN
INDICATIONS.
IT'S HARD TO SIFT THROUGH ALL
THE LITERATURE AND FIND OUT
WHICH ONE IS GOING TO BENEFIT
FROM THE SMALL MOLECULE AND WE
STUMBLED ON TO A COUPLE.
THE NEXT THING THE CHALLENGE WE
HAD TO DO IS FIND PEOPLE TO TEST
THE SMALL MOLECULE WITH AND NO
ONE HAS FUNDING SO IN THEORY
YOU'D NEED TO HAVE SOME
MECHANISM IF YOU CAN PAIR UP
WITH AN ORPHAN INDICATION
CHEMIST AND FIND A BIOLOGIST AND
HAVE PROOF OF CONCEPT FIRST
PASSES.
WE'VE HAD TO DO IT ON OUR OWN
DIME AND NOW PUTTING IN AN SBIR
GRANT TO PAVE THE NEXT STEP.
PROVING IT WORKS IS CRITICAL.
YOU HAVE TO PAIR UP AND FIND A
MECHANISM TO ADVERTISE TO THE
ACADEMIC AND PAIR THEM AND THEN
TO HAVE FUND IING AND TO FIND
VENTURE CAPITAL YOU HAVE
PLATFORM DATA THAT SAYS YOUR
SMALL MOLECULE IS WORK IN YOUR
ORPHAN INDICATION.
AND YOU HAVE TO APPLY FOR ORPHAN
MOLECULE AND DISEASE AND JUST
TALKED TO SOMEBODY WHO WANTED
$50,000 TO DO THAT AND ONCE YOU
GET THE DESIGNATION AGAIN
VENTURE CAPITAL MONEY OPENS UP
TO GO FORWARD AND DEVELOP THIS
BECAUSE THEY SEE THE BENEFIT
YOUR TARGET YOU'RE GOING AFTER
HAS THIS LONG-TERM FINANCIAL
BENEFIT OF BEING DESIGNATED AS
AN ORPHAN INDICATION AND GOING
BACK TO THE SUGGESTION OF THESE
STAKEHOLDERS.
WE'VE BEEN DESPERATELY TRYING TO
REACH OUT TO STAKEHOLDERS FOR
THE PARTICULAR ORPHAN
INDICATIONS WE'RE INTERESTED IN
TO HAVE THEM HELP US FIND
VENTURE CAPITAL.
THAT'S WHAT'S KILLING US.
WE CAN'T FIND IT TO GO TO THE
NEXT STEP.
WE ARE LOOKING AT FAMILY
INVESTORS AND PEOPLE WITH MONEY
TO HELP US GET OVER THE HUMP.
THE SMALL STAKEHOLDERS GROUPS,
IF YOU CAN HELP THEM UNDERSTAND
THE SYSTEM AND MAYBE JS THEM HOW
THEY CAN GET FUNDING AND MAYBE
NOT JUST GET FUNDING BUT PARTNER
WITH SCIENTIST TO GO OUT TO THE
LARGE INVESTMENT FIRMS,
SOMETIMES THAT CAN HELP THE
STAKEHOLDER.
SOMETIMES THEY LIKE HEARING
ABOUT THE STAKEHOLDERS AND
SOMETIMES THEY ARE ALL BUSINESS
AND DON'T GIVE A DAMN ABOUT THE
BOTTOM LINE.
I REMEMBER GOING TO THE
SYMPOSIUM ON CANCER AND TALKED
ABOUT THEIR PERSONAL EXPERIENCE,
THE STORIES MOTIVATE BASIC
SCIENTISTS TO DO MORE.
I DON'T KNOW HOW THAT PLAYS IN.
I'M GIVING IDEAS.
I'M NOT SURE I HAVE SOLUTIONS
BUT TRYING TO GIVE A PERSPECTIVE
FROM MY PERSPECTIVE OF HAVING
DEVELOPED SMALL MOLECULES AND
KNOWING V.C. WANTS ORPHAN
INDICATION THAT SOMETHING WORKS.
HOW DO WE PAIR UP SOMEONE LIKE
MYSELF TO THESE INCREDIBLE
GROUPS AS WELL AS ANIMAL MODELS
TO GET THE PRELIMINARY DATA TO
INSPIRE V.C. TO GIVE MORE MONEY
TO GO FORWARD.
I HOPE I HAVEN'T CONFUSED YOU
BUT TRYING TO GIVE MY
PERSPECTIVE ON THE CHALLENGES
I'VE PERSONALLY HAD THE LAST
FIVE OR SIX YEARS TRYING TO GET
MY SMALL MOLECULES TO SOMEONE
WHO CAN HELP.
>>RIGHT.
WE WANT TO UNDERSTAND HOW TO
BRING THE PARTIES TOGETHER AND
PROVIDING THE SUPPORT TO DO
THAT.
IN TERMS OF IDENTIFYING THE
ORPHAN INDICATION AND TRANSLATOR
ISN'T QUITE READY FOR PRIME TIME
YET.
HOPEFULLY TRANSLATOR WILL HELP
AT SOME POINT.
THERE USED TO BE LITERALLY A
SPREAD SHEET AVAILABLE THROUGH
FDA AND IF THAT'S STILL
AVAILABLE WE CAN DIG THAT OUT.
I THINK WE SHOULD BE ABLE TO
LINK TO IT.
>>WHAT TED SAID RESONATES TO
FINDING WAYS WHERE YOU CAN
CONVENE ALL THE GROUPS TOGETHER
EVEN IF YOU'RE KEEPING TRACK OF
WHAT ARE THE AVAILABLE RESOURCES
AROUND THE RARE DISEASE MODEL OR
THE SMALL MOLECULE GROUPS EXIST
AND I KNOW THEY EXIST BUT I
THINK THEY'RE GOOD POINTS.
>>I WANT TO GIVE CREDIT TO
STEVE WHO RECOGNIZED THERE IS
THIS NEED AND MAYBE BEFORE I GO
TO MATIAS TO SEE IF SHE WANTS TO
RESPOND AS WELL.
>>WE GET TO THE MATCH MATCHING
WE CAN THEORETICALLY DO WITH THE
GROUPS THAT HAVE THE MODELS AS
WELL AS WITH THE V.C. AND I
WORKED WITH THE OFFICE OF
STRATEGY ALLIANCE ON IS A WORK
SHAPE SPECIFIC TO PAIN AND
BRINGING V.C. AND INDUSTRY
EXPERTS INTERESTED IN PAYING AND
WE'RE PUTTING THEM IN FRONT OF
THE SBIR AWARDEES THAT HAVE
PROJECTS THERE.
DOING A SIMILAR WORKSHOP WITHIN
THE RARE DISEASE SPACE IS OF
INTEREST AND I TALK TO THE GROUP
ABOUT THAT NOW AND THINK WE CAN
MOVE THAT FORWARD.
>>THERE'S SWISS SHOWCASE.
PEOPLE WHO HAVE PROJECTS AND
THEY HAVE A HOOK.
IT'S GREAT.
IT GETS IN FRONT OF THE FUNDERS
AND IT MAKES A DIFFERENCE.
>>THANK YOU.
MATIAS.
>>WHAT WE HAVE IS 80% OF THE
PEOPLE WE KNOW DON'T KNOW WHAT
A GENETIC LESION DOES AND WE'RE
ACTIVE IN MAPPING THE ASSOCIATED
PATHWAYS ACCORDING TO
DYSFUNCTION.
WE HAVE COMPOUNDS TO INTERFERE
WITH THE BIOLOGICAL PROCESSES.
SOMETIMES WE KNOW THEM,
SOMETIMES WE DON'T YET WE CAN AT
LEAST GET A MECHANISTIC
ASSOCIATION AND WE HAVE DONE
THAT EFFECTIVELY FOR KNOWN
MOLECULE PATHS.
AND YOU HAVE TO GET A MODEL
SYSTEM WITHIN THE CELL-BASED
ASSAYS TO WORK WITH STAFF.
THERE'S CHIPS AND YOU CAN PUT
THEM IN AND CLEARLY THEY'LL NEED
TO BE FEATURES IN HERE AND YES,
WE ARE DOING THAT IN OUR END
TOGETHER WITH THE CHIP
EFFECTIVELY AND AND WE PROFILE
TO A PHASE 2 TRIAL DOING EXACTLY
THAT ELEMENT.
CRITICAL INSTRUMENT HERE AND
WHERE WE INITIATED
PRE-COMPETITIVE CONSORTIUM TO
MAP TO DISEASE AND WHERE THEY
CHANNEL TOGETHER AND WOULD CAN
IMPACT MORE THAN ONE OF THE
DISEASES ALONE.
AND MAP AGAINST A HUMAN DISEASE
MAP AND IT'S UNDER THE BIO
INFORMATICS.
IF YOU HAVE THE MODEL SYSTEM OR
TERMS THEN YOU KNOW WHICH MODEL
SYSTEM MAPS INTO WHICH HUMAN
DISEASE OR WHICH HUMAN DISEASE
MAPS TO WHICH MODEL SYSTEM.
AND WE NEED TO ASK WHICH HUMAN
DISEASE IS LINKED TO THE MODEL
SYSTEM TO REACH OUT AND CONTACT
THE RARE DISEASE COMMUNITY AND
WHAT IS INQUIRED IS THE FEE
PHENOTYPING.
THE MODEL SYSTEMS AND GETS BACK
TO DATA SHARING METHODS.
AND YOU GET THE MODEL SYSTEMS
CHARACTERIZED IN A WAY TO BE
USEFUL ABOVE AND BEYOND THE
MOLECULES TARGETED TO THIS
APPROACH.
AND IN THE RARE DISEASE SPACE
IT'S EXACTLY WHAT WE DID.
WE HAD HALF A DOZEN MODEL
SYSTEMS ON SINGLE CELL LEVEL
PHENOTYPE AND WE ARE ALIGNING
THE PATHWAYS AGAINST COMPANIES
WANTING TO REPURPOSE INTO THE
SPACE AND KNOW WHAT IS LIVING
WHERE TO BRING THEM TO TARGETED
THERAPIES AND WE PUBLISHED INTO
MAPPING AND WE LOOK AT
PHENOTYPING IN A WAY TO GLOBALLY
MAP AGAINST THIS AND PHENOTYPE
IN A WAY THAT OTHER PATHWAYS
COULD BE PROJECTED BACK TO THE
HUMAN DISEASE SPACE.
>>YEAH, NO.
THAT'S A GREAT SUGGESTION.
I LOVE THAT.
IT'S SOMETHING I HADN'T THOUGHT
ABOUT AND SO YEAH, THAT SOUNDS
REALLY GOOD.
>>FIRST, I AGREE WITH MARSHALL
THERE'S MANY PATIENT
ORGANIZATIONS FUNDING THIS WORK
FOR A LONG TIME AND SAVVY IN THE
SPACE AND HAVE LEARNED A LOT OF
LESSONS IN THE SPACE AS WELL AS.
WE HAVE INSIGHTS AND HIND SIGHT
TO SHARE.
I MIGHT SUGGEST TALKING TO THEM
NOT JUST ABOUT BEST PRACTICES
BUT IN HINDSIGHT WHAT WE MAY
HAVE DONE NEWER TO THE SPACE AND
RELATIONSHIP.
THE OTHER IS TO TEST POINTS AND
TO STEVEN'S COMMENTS AROUND THE
MATCHING AND MATCH MAKING.
THERE ARE SOME ORGANIZATIONS
THAT HAVE SET UP FORMALIZED
PROGRAMS IN THE SPACE AND I PUT
ONE OF THEM IN THE CHAT HERE BUT
WHEN I WAS WITH THE MUSCULAR
DYSTROPHY ASSOCIATION WE SAID UP
MVP WHICH HAS BEEN SUCCESSFUL IN
FINDING VENTURE PHILANTHROPISTS
EARLY ON AND I RECOMMEND TALKING
TO MDA AND SURE YOU KNOW THE
SCIENTIFIC LEADERS THERE.
SO THERE'S ORGANIZATIONS THAT
HAVE TACKLED SOME OF THIS.
MAYBE TO THE POINT OF
PARTNERSHIP AND WORKING WITH THE
COMMUNITY.
THESE ARE THINGS THE RARE
DISEASE COMMUNITY CARES DEEPLY
ABOUT AND I AGREE WITH MARSHAL
WORKING WITH OUR COLLABORATIONS
AND ORGANIZATIONS AND
ADVERTISING THIS AND KNOWING
WHERE THE OPPORTUNITIES ARE KEY.
YOU HAD A BULLET AROUND HOW THAT
ENGAGEMENT WILL BE CRITICAL AND
I AM SURE IT'S JUST FOR THE SAKE
OF SPACE ON THE SLIDE THE BULLET
HAD YOUR FIRST PRIORITY, WHICH I
APPRECIATED WAS UP THERE WOULD
BE ENGAGING WITH I THINK
CLINICAL EXPERTS AND PATIENTS
AND PATIENT ADVOCACIES AND I
WANT TO POINT OUT THOSE ARE
THREE DISTINCT STAKEHOLDER
GROUPS WITH DISTINCT PURPOSES
AND APPROACH THEM INDIVIDUALLY.
ALL THIS IS WONDERFUL AND THANK
YOU FOR PRIORITIZING THIS.
>>THIS IS AN INTERESTING
CONVERSATION WITH COUNCIL
MEMBERS.
I APPRECIATE THE COMMENTS AND
SUGGESTIONS.
AND TO BUILD A LITTLE BIT ON
WHAT'S ON ONE OF YOUR SLIDES TO
PUT PATIENTS BACK BY REQUIRING
PARTNERSHIPS AND MENTIONED THREE
STAKEHOLDERS INCLUDING PATIENTS
AND PATIENT ADVOCATES.
AND PUSH BASIC SCIENTISTS TO
THINK MORE INTENTIONAL ABOUT
STUDIES THAT HAVE MORE RELEVANCE
TO THE PATIENT POPULATION IN
QUESTION.
AND WE MOVE TO THE TRANSLATIONAL
SCIENCE AND RESEARCH SPACE.
I'M GLAD YOU INCLUDED THAT
BECAUSE IT WOULD BE GREAT TO
HAVE A SENSE FROM THE
RESEARCHERS THAT ARE GOING TO
APPLY FOR THIS MECHANISM TO
REALLY BECOME TRANSLATIONAL
RESEARCHERS.
AND IF THEY'RE PUSHED TO
ARTICULATE HOW THAT'S GOING TO
HAPPEN.
FOR EXAMPLE, I WOULD LOVE TO SEE
THE DISSEMINATION PLAN WORKING
WITH THE PATIENTS AND FAMILIES
THEMSELVES HOW TO BRING THAT TO
THEIR ATTENTION.
BECAUSE, I AM NOT AN EXPERT IN
RARE DISEASES BUT I ANTICIPATE
THERE'S TREMENDOUS DISPARITIES
THERE AS TO WHO BENEFITS
SPECIFICALLY FROM THE DRUG
DEVELOPMENT.
>>I THINK WE WANT TO ADDRESS
WHERE THE RESEARCHER DOESN'T
REALIZE THEY MISSED THE
OPPORTUNITY AND MAKE IT MORE
OBVIOUS FOR THEM.
THERE'S OPPORTUNITY I'VE NEVER
MED A PRE-CLINICAL SCIENTIST WHO
TURNED DOWN THE OPPORTUNITY TO
HAVE WHAT THEY'RE DOING BECOME
SOMETHING THAT IMPACTS.
>>NOBODY WOULD SAY, NO, NO, NO,
I WANT TO GO BACK TO DOING MY
THING WHERE IT DOESN'T HAVE
ANYTHING TO DO WITH THE REAL
WORLD.
AND FROM A PRACTICAL STANDPOINT
IT'S NOT NECESSARY LE THE WAY
THE RESEARCHERS ARE GOING TO
SPEND THEIR LIFE TO TRY TO GET
TO THAT POINT.
WHEN WE SHOW THEM THERE'S AN
OPPORTUNITY AND OBVIOUSLY IT
WON'T FIT FOR ANYBODY, ANYWAY.
IN THE CASES WHERE IT DOES TO
LOWER THE ENERGY BARRIER IS WHAT
WE WANT TO BE ABLE TO DO FOR
THEM.
AND THEN HOPEFULLY THEY'LL TELL
THEIR FRIENDS AND WE'LL GET MORE
AND MORE SCIENTISTS THAT WAY.
>>IF YOU CAN IDENTIFY THIS IS
THE MODEL SYSTEM YOUR MOLECULE
LINKS TO, THIS IS A DISEASE
WHERE THE MOLECULE LINK TO
THEY'LL GET IT AND WILL ENGAGE.
IF YOU COME WITH, YOU KNOW, AN
ABSTRACT PHENOTYPE AND WITH HAVE
THE TOOLS NOW.
WE HAVE PHENOTYPES AND YOU HAVE
A FORMAT TO LINK INDIVIDUAL
PATIENT LEVEL ASSAYS TO THOSE
MOLECULES.
THAT'S A GAME CHANGER.
IF YOU GET YOUR SYSTEM
SOPHISTICATED ENOUGH AND HUGE
STEPS HAVE BEEN MADE, WE'RE
STILL NOT AT THE STAGE TO FULLY
REPLACE THE ANIMAL MODEL SYSTEM.
THAT'S THE BIGGEST POINT AND
WHERE I THINK IT FITS NICELY
INTO AND IF YOU CAN CORRECT THE
MODEL SYSTEMS AND IDEALLY NOT
ONE-OFFS ONLY, IF YOU ESTABLISH
AN APPROACH OR PHILOSOPHY
BECAUSE YOUR PHILANTHROPICS ARE
LEARNING AND WE'LL FUND YOU BUT
AT LEAST GENERATE DATA SO THE
NEXT MOLECULE HAVE A MODEL
SYSTEM.
>>MAKE YOU'RE COMMENT AND WE'LL
PROCEED WITH OUR VOTING ON THE
CON SET.
>>THANK YOU, ANN.
I WANT TO PUSH A LITTLE BIT
ABOUT WHAT I THINK WOULD BE
GREAT IN THE CONTEXT OF
TRANSLATIONAL SCIENCE.
WOULDN'T THE BE GREAT TO HAVE A
MEASURE OF SUCCESS WHEN THE
DRUGS ARE DEVELOPED ONCE
PATIENTS ARE IDENTIFIED WITH THE
RARE DISEASES AND DRUGS
DEVELOPED ARE FOUND TO BE
EFFECTIVE AND IDENTIFIED AND WE
SHORTEN THE TIME BETWEEN WHEN
THEY ARE DIAGNOSED TO RECEIVING
THE TREATMENT.
IT'S IMPORTANT TO HAVE VISIONS
LIKE THAT WHEN PATIENTS BENEFIT
FROM THESE DEVELOPMENTS.
>>GREAT POINT.
IF WE CAN SHORTEN THE DIAGNOSTIC
ODYSSEY IT WILL MAKE A HUGE
DIFFERENCE.
>>IT FITS AND KEEP PUSHING.
THAT'S WHAT WE WANT TO DO.
GO AHEAD AND START WITH THE
CONCEPT VOTE.
THANK YOU.
ALL RIGHT.
IF THERE'S NO FURTHER
DISCUSSION, COULD WE HAVE A
MOTION TO APPROVE THE CONCEPT?
>>MOVE.
>>SECOND.
>>ALL IN FAVOR?
ANY OPPOSED?
ANY ABSTENTIONS AND NOT
SURPRISINGLY THE CONCEPT IS
APPROVED.
WE'LL NOW MOVE TO A BREAK --
>>MAY I INTERRUPT QUICKLY.
I WANTED TO PULL OUT A COMMENT
ANNIE KENNEDY MADE WE DIDN'T GET
A CHANCE TO HIGHLIGHT ABOUT THE
STRATEGIC PLAN AND HOW TO INFUSE
THE 21st CENTURY CURES
DISCUSSION.
I WANTED TO GIVE ANNIE AN
OPPORTUNITY BEFORE WE TAKE A
BREAK TO CAPTURE THAT CORRECTLY.
WOULD YOU MIND HIGHLIGHTING THAT
COMMENT IN THE CHAT.
>>I'LL MAKE IT QUICK TO GET A
BREAK.
I WAS SUGGESTING AND REMINDING
PEOPLE AND PROBABLY A LOT OF
PEOPLE HERE WHO PARTICIPATED BUT
AS THE COMMUNITY AND CONGRESS
PREPARES FOR THE DRAFTING OF THE
21st CENTURY CURES LEGISLATION,
THERE WERE ROUNDTABLES CONVENED
THROUGHOUT THE NATION AND THERE
WAS A LOT OF WONDERFUL INSIGHT
AND MULTI-STAKEHOLDER
ROUNDTABLES.
THEY HAD PATIENTS AND PATIENT
ADVOCATES AND CLINICIANS AND
BENCH SCIENTISTS AND
TRANSLATIONAL SCIENTISTS AND
THOSE WHO ARE CLINICAL TRIALISTS
TALKING ABOUT OPPORTUNITIES FOR
INNOVATION WITHIN OUR ECO
SYSTEMS.
SO MY SUGGESTION WAS MAYBE TO GO
BACK AND LOOK AT SOME OF THE
PUBLICATIONS THAT CAME FROM THAT
AND SOME OF THE TRANSCRIPTS ALL
OF WHICH ARE PUBLIC AS WE THINK
ABOUT AN APPROACH TO THE
STRATEGIC PLANNING FOR NCATS
BECAUSE THERE'S DIRECT RELEVANCE
THERE.
THERE'S WONDERFUL THINGS TO MOVE
FORWARD IN 21st CENTURY CURES
AND THINGS THAT WEREN'T
APPLICABLE TO THE LEGISLATION
THAT MAY BE APPLICABLE TO THE
NCATS STRATEGIC PLAN AS YOU
THINK OF HOW TO APPROACH THIS.
>>BRILLIANT IDEA.
I WANTED TO MAKE SURE WE ALL
HEARD IT TOO IN CASE SOMEBODY
DIDN'T SEE IT IN THE CHAT.
THANK YOU FOR BRINGING THAT UP.
IT'S FANTASTIC.
WE'LL TAKE A LOOK AND SEE HOW TO
DO THAT.
ANNA, THANK YOU.
SORRY ABOUT THAT.
PLEASE GO AHEAD AND WE'LL
CONVENE FOR A BREAK.
>>GOOD AFTERNOON AND WELCOME
BACK TO THE NEXT PART OF THE.
SESSION.
I'M THE ACTING DEPUTY DIRECTOR.
JONI WAS CALLED AWAY AND WILL BE
BACK SHORTLY.
IN THE MEANTIME IT'S MY GREAT
PLEASURE TO INTRODUCE OUR GUEST,
DR. MIKE LAUER.
THE NIH DEPUTY DIRECTOR FOR
EXTRAMURAL RESEARCH.
HE SERVES AS THE PRINCIPAL
SCIENTIFIC DIRECTOR AND RELATE
TO THE SUBSTANCE, QUALITY AND
EFFECTIVENESS OF THE NIH
EXTRAMURAL RESEARCH PROGRAM AND
ADMINISTRATION.
TODAY MIKE WILL BE SHARING AN
UPDATE ON THE NIH DATA SHARING,
DATA MANAGEMENT AND SHARING
POLICY.
WELCOME, MIKE.
>>THANK YOU FOR THE OPPORTUNITY
TO BE HERE AND TO SPEAK WITH YOU
ABOUT THE DATA MANAGEMENT AND
SHARING POLICY.
I'VE GIVEN VARIOUS VERSIONS OF
THIS TALK BEFORE AND ALWAYS
TALKED ABOUT SOMETHING THAT WAS
GOING TO HAPPEN SOME TIME IN THE
FUTURE.
THE FUTURE IS NOW AND WE'RE AT
THE POINT WHERE THE POLICY HAS
BEEN IMPLEMENTED.
NOW DATA SHARING AND THE AND JOE
BIDEN HELPED LEAD THE
DEVELOPMENT OF THE CANCER MOON
SHOT AT NIH AND TALKED ABOUT
DATA SHARING AS A CORE COMPONENT
AND BY 2016 BY OVERWHELMING
BIPARTISAN MAJORITIES THE 21st
CENTURIES CURES ACT WAS SIGNED
AND PASSED BY PRESIDENT OBAMA.
IN THAT LAW THERE WAS A
STATEMENT THE NIH MY REQUIRE
RECIPIENTS OF AWARDS TO SHARE
SCIENTIFIC DATA TO THE EXTEND
AND THE IDEA IT'S IMPORTANT IS
IN STATUTE.
AND THE POLICY HAD BEEN
IMPLEMENTED IN 2003 AND REQUIRED
MANAGEMENT OF AWARDS FOR DIRECT
COSTS IN A GIVEN YEAR.
THERE WERE OTHER POLICIES PUT
INTO PLACE PERHAPS BEST KNOWN AS
THE 2014 NIH GENOMIC DATA
SHARING POLICY AND THERE'S BEEN
A NUMBER OF AND THE CURRENT
POLICY JUST IMPLEMENTED POLICY
GOES BACK MANY YEARS.
THE AGENCY HAS ISSUED TWO RFIs
REQUEST FOR INPUT AND ONE RFC,
REQUEST FOR COMMENTS AT VARIOUS
POINTS ALONG THE WAY BACK IN
2016, 2018 AND 2019.
THERE WAS A CONSULTATION WITH A
NUMBER OF OTHER UNITS WITHIN THE
U.S. GOVERNMENT AS WELL AS
TRIBAL CONSULTATION.
THE POLICY ITSELF WAS RELEASED
IN OCTOBER OF 2020 ALONG WITH
SUPPLEMENTARY DOCUMENTS.
AND IT WOULD BE INITIALLY
IMPLEMENTED IN JANUARY OF 2023
WHICH IS WHERE WE ARE NOW.
AND IT PRODUCES REPRODUCIBLE
RESEARCH THROUGH A VARIETY OF
WAYS.
THERE WAS SOMETHING DONE BY THE
CENTER BY OPEN SCIENCE.
AND THEY IDENTIFIED 193
EXPERIMENTS PUBLISHED IN HIGH
IMPACT PAPERS AND WANTED TO SEE
WHETHER OR NOT THEY COULD
REPLICATE THE RESULTS.
THEY COULD DO SOME AND NOT
OTHERS.
IN MANY CASE THE EFFECT SIZES
AND MAYBE THE EFFECT SIZES
TURNED OUT TO BE SMALLER THAN
INITIALLY REPORTED.
ALL THAT IS INTERESTING BUT FOR
THE PURPOSES OF THIS DISCUSSION,
ONE OF THE ASPECTS OF THE
PROJECT IS TO REACH OUT TO THE
AUTHORS TO THE ORIGINAL PAPERS
AND GET ORIGINAL DATA IN
DESIGNING THE REPLICATION
EXPERIMENTS.
DESPITE CONTACTING THE AUTHORS
OF THE ORIGINAL PAPERS WE WERE
UNABLE TO OBTAIN THE DATA FOR
68%.
YOU MAY SAY IN THAT CASE THE
AUTHORS NEVER PROMISED TO SHARE
THE DATA ANY WAY.
THIS IS FROM A PAPER IN EUROPE
THAT WAS REPORTED OUT EIGHT OR
NINE MONTHS AGO.
WHAT THEY DID WAS THEY REACHED
OUT TO AUTHORS WHO HAD PUT A
DATA AVAILABILITY STATEMENT IN
THE PAPER.
AND THERE WAS A DATA
AVAILABILITY STATEMENT THAT SAID
SOMETHING LIKE TE DATA WILL BE
MADE AVAILABLE.
AND THEY WERE ONLY TO GET DATA
FROM 7% OF THE AUTHORS.
SO BOTH OF THESE STORIES ARE
RATHER DISCOURAGING.
HERE'S A STORY ENCOURAGING.
AND I SAW THEY HAD A DATA
REPOSITORY BEFORE THEN BUT THEY
TOOK IT TO A HIGHER LEVEL AND
MADE IT MORE SYSTEMATIZED AND
COLLECTED DATA FROM A VARIETY OF
DIFFERENT KINDS OF STUDIES.
AMONG THE STUDIES THEY COLLECTED
DATA FOR WERE CERTAIN CLINICAL
TRIALS THAT WERE FUNDED BY THE
INSTITUTE.
THEY WANTED TO SEE WHAT HAPPENED
TO THE DATA WE'RE NOW BEING MADE
AVAILABLE TO OUTSIDE RESEARCHERS
AND FOUND FOR 100 OF THE
CLINICAL TRIALS AT THE TIME OF
THE STUDY DATA WERE REQUESTED
FOR THEM AND FROM THE TIME TRAIL
DATA WERE MADE UNTIL THE FIRST
REQUEST WAS 235 DAYS.
LESS THAN NINE MONTHS.
AN THERE WERE CITATION VALUES ON
PAR WITH NHLBI FUNDED PAPERS.
I WASN'T INVOLVED IN THE STUDY
OR AN AUTHOR OF THE STUDY BUT
WHEN THE DATA REPOSITORY WAS SET
UP AND I'M A CARDIOLOGIST BY
BACKGROUND AND I REQUESTED DATA
FROM AN EPIDEMIOLOGY STUDY AND
TO MY AMAZEMENT IT WASN'T THAT
HARD.
AND I WAS ABLE TO GET THE DATA
AND LOOK AT THE DATA AND
UNDERSTAND IT AND WE HAVE PAPERS
FROM THE FIRST DATA REQUEST AND
DID THAT FOR AT LEAST ONE OTHER
STUDY WHICH LED TO PAPERS BEING
PUBLIS
PUBLISHED AND I WORKED WITH SOME
OF THE ORIGINAL SCIENTISTS.
THAT'S GOOD NEWS.
AND IT'S PORE -- MORE LIKELY TO
BE TRUSTED FROM AN INDEPENDENT
COMMITTEE AND LESS SO FUNDED BY
THE FEDERAL GOVERNMENT AND NOT
LUCKY TO BE TRUSTED FUNDED BY
INDUSTRY.
AND NOW TO THE POLICY ITSELF.
IN SOME RESPECTS IT'S SIMPLE.
WHEN AN APPLICATION IS SUBMITTED
TO THE NIH IT HAS TO BE
COMPANIED BY A DATA MANAGEMENT
AND SHARING PLAN.
THEN ONCE THAT PLAN IS APPROVED,
THEN THERE NEEDS TO BE
COMPLIANCE WITH THE PLAN.
AND THIS POLICY REQUIRING DATA
MANAGEMENT AND SHARING PLANS
BECAME EFFECTIVE YESTERDAY.
SO IT'S EFFECTIVE FOR ALL
APPLICATIONS OR RELEVANT
APPLICATIONS AND SUBMITTED
JANUARY 25, 2023 AND LATER.
NOW, HAVING SAID THIS WAS FAIRLY
SIMPLE, THERE ARE A NUMBER OF
DETAILS.
ONE QUESTION IS WHAT EXACTLY DO
WE MEAN BY DATA.
OF COURSE WE HAVE A DEFINITION
FOR THAT.
WE DEFINE IT AS RECORDED FACTUAL
MATERIAL TO VALIDATE AND
REPLICATE FINDINGS THEY'RE USED
TO SUPPORT SCHOLARLY
PUBLICATIONS.
IT DOESN'T INCLUDE LAB NOTEBOOKS
OR OBJECTS OR CASE REPORT FORMS
OR PRELIMINARY ANALYSES.
AND ONE INTERESTING EXERCISE WE
WENT THROUGH WHEN WE OBTAINED
SHARED DATA FROM NHLBI WE
ATTEMPTED TO REPLICATE THE
FINDINGS REPORTED IN THE
ORIGINAL PAPERS.
IT WASN'T WE HAD ANY REASON TO
BELIEVE THERE WERE PROBLEMS BUT
IT WAS OUR WAY OF DETERMINING
WHETHER OR NOT WE ACTUALLY
UNDERSTOOD THE DATA WE HAD.
AND TO SEE IF WE CAN COME UP
WITH THE SAME NUMBERS THAT
APPEARED IN THE PAPERS.
THAT ALONE WAS ALSO A REALLY
GOOD EXERCISE IN AND OF ITSELF.
THIS APPLIES TO RESEARCH THAT
GENERATES DATA.
TRAINING A FELLOWSHIP
CONSTRUCTION, CONFERENCE AWARDS
DO NOT ARE NOT APPLICABLE AND
DATA SHOULD BE SHARED NO LATER
THAN THE TIME OF PUBLICATION AND
FOR DATA THAT ARE NOT
ACCOMPANIED BY PUBLICATION WOULD
BE THE END OF THE AWARD.
THE GENERAL DEFAULT VIEW IS DATA
SHARING SHOULD BE MAXIMIZED.
ALL DATA NEEDS TO BE MANAGE D NT
ALL DATA NEEDS TO BE SHARED AND
WE'RE ASKING FOR PLANS THAT
DESCRIBE HOW THE DATA WILL BE
MANAGED AND SHARED IN A
RESPONSIBLE WAY AND ONE OF THE
KEY POINTS IS THAT THIS IS A
PROSPECTIVE PROCESS.
WE'RE ASKING FOR PLANS AT THE
TIME OF APPLICATION.
NOT AFTER THE DATA HAS BEEN
GENERATED BUT BEFORE THE DATA IS
GENERATED HOW ARE YOU THINKING
ABOUT IT.
NOT ALL DATA NEEDS TO BE SHARED
AND THERE ARE JUSTIFIABLE,
ETHICAL, LEGAL AND TECHNICAL S
AND THERE MAY BE PRIVACY OR
SAFETY ISSUES OR RELEVANT STATE,
LOCAL TRIBAL LAWS OR FEDERAL
LAWS EVEN.
THERE MAY BE OTHER KINDS OF
RESTRICTIONS WITH THIRD PARTIES.
THIS IS ALL UNDERSTOOD.
THIS WOULD BE PART OF THE DATA
MANAGEMENT SHARING PLAN.
THERE MAY BE A STATEMENT THAT
CERTAIN DATA WILL NOT BE SHARED
FOR THE FOLLOWING REASONS AND
THAT'S A CONVERSATION THE
APPLICANT WOULD HAVE WITH NIH.
REASONS NOT TO IS THE DATA ARE
SMALL OR UNINTERESTING OR WE
THINK THE DATA ARE
UNINTERESTING.
OR NOT THOUGHT TO HAVE
SUITABILITY AND THERE'S ISSUES
WITH TRIBAL SOVEREIGNTY AND THE
SBIR AND NSTR PROGRAM.
TO HELP THE COMMUNITY ADDRESS
THIS LAST APRIL WE RELEASED A
NEW WEBSITE CALLED
SHARING.NIH.gov AND SERVE AS A
CENTRAL SOURCE OF GUIDANCE.
THERE'S INFORMATION ABOUT MODEL
ORGANISMS AND RESEARCH TOOLS.
IT'S A HIGHLY DYNAMIC LIVING
WEBSITE AND THIS PARTICULAR
PICTURE I TOOK A COUPLE DAYS AGO
BECAUSE IT SAYS THERE'S FEATURED
NEWS AND EVENTS AND CHECK OUT
OUR DMS PLANT AND VIEW MORE AND
I'LL TALK ABOUT THAT IN A
SECOND.
THERE'S RESOURCES FOR DIFFERENT
KINDS OF DATA.
DIFFERENT KINDS OF SHARING I
SHOULD SAY.
SCIENTIFIC DATA, GENOMIC, MODEL
ORGANISMS AND RULES RELATED TO
CLINICAL TRIALS.
WE HAVE -- I COULD CALL IT A
WIZARD.
WHICH POLICIES APPLY TO MY
RESEARCH.
LEADS YOU THROUGH A SERIES OF
QUESTIONS AND THIS HELPS YOU
FIGURE OUT WHAT IS RELEVANT TO
AN APPLICATION YOU MAY BE
SENDING IN.
WE HAVE RESOURCES ON DIFFERENT
APPROACHES TO SHARING DATA ON
REPOSITO
REPOSITORIES HOW IT WRITE A PLAN
AND BUDGET.
WE RECOGNIZE THIS IS NOT FREE.
WHEN THE ORIGINAL POLICY WAS
RELEASED IN OCTOBER, 2020 WE
INCLUDED SUPPLEMENTARY DUMES AND
WE TALKED ABOUT A SHARING PLAN
AND THAT HAS NOT CHANGED SINCE
THAT TIME.
WE HAVE PUT OUT ADDITIONAL
RESOURCES ABOUT WHAT TYPES OF
THINGS WE'RE LOOKING FOR IN A
DATA MANAGEMENT SHARING PLAN AND
THEY ARE TAKING STRAIGHT FROM
THE DOCUMENT RELEASED AND WE
ASKED ABOUT DATA TYPE AND
RELATED TOOL, SOFTWARE AND CODE
THAT MAY BE AVAILABLE AND WHAT
STANDARDS ARE IN PLACE, WHAT
STEPS ARE GOING TO BE TAKEN TO
PRESERVE THE DATA AND HOW WILL
ACCESS OCCUR AND WHAT TIME LINES
ARE YOU ANTICIPATING AND ARE
THERE ACCESS IN CONSIDERATIONS
FOR EXAMPLE, WILL THE DATA BE
MADE SIMPLY PUBLICLY AVAILABLE
TO ANYONE WHO WANTS IT AND N
HANES AND GO ON THE WEBSITE AND
DOWNLOAD THE DATA AND YOU DON'T
HAVE TO TALK TO ANYBODY OR MAYBE
LIKE THE NHLBI DATA REPOSITORY
WHERE I HAD TO ASK FOR THE DATA
AND GOT IT AND THINK ABOUT DATA
THAT'S SENSITIVE OR IDENTIFIABLE
LIKE GENOMIC DATA.
WE HAVE CONTROLLED ACCESS WHERE
MANY STEPS HAVE TO BE TAKEN
BEFORE ONE CAN GET ACCESS TO THE
DATA.
AND THEN WE ALSO ASK HOW THE
DATA MANAGEMENT WILL BE
OVERSEEN.
ONE ONE WE HEARD FROM THE
COMMUNITY A LOT WAS A DESIRE TO
SEE SAMPLE PLANS.
THIS IS A BIT OF A MIXED BAG
HERE.
ON THE ONE HAND SAMPLE PLANS,
HOPEFULLY WILL HELP PEOPLE WRITE
THEIR OWN PLANS AND WRITE DECENT
PLANS BUT ON THE OTHER HAND WE
DON'T WANT TO SAY IT'S THE ONLY
ACCEPTABLE PLAN OUT THERE.
AND THESE ARE SOME SAMPLE PLANS
WE HAVE ALREADY I ANTICIPATE
OVER TIME WE'LL POST MORE SAMPLE
PLANS AND I'M ANTICIPATING AND
WITH THE PERMISSION OF THE
SCIENTISTS, WE'LL POST THEIR
PLANS HOPEFULLY APPROPRIATELY
REDACTED AND POST THE PLANS AS
SAMPLES OTHERS CAN USE TO PUT
THE PLAYERS TOGETHER.
WE PROVIDE GUIDANCE IN SELECTING
THE REPOS -- REPOSITORY IS
EASY.
THE FOA MAY SAY YOU MUST STATE
DATA WILL BE DEPOSITED IN THE X,
Y, Z REPOSITORY.
IT MAKES THE DECISION EASY.
THERE'S MANY KINDS OF
REPOSITORIES OUT THERE.
SOME ARE GENERALIZED AND
SPECIALIZED.
WE HAVE A LIST OF NIH
REPOSITORIES MANAGED BY OUR
INSTITUTES.
MOST OF THESE ARE SPECIALIST
REPOSITORIES.
AN EXAMPLE OF ONE DB GAP IS BEST
KNOWN AND BIO LINK.
IT'S THE REPOSITORY NHLBI
MAINTAINS AND THE ONE AS AN
EXTERNAL EXTRAMURAL INVESTIGATOR
TOOK ADVANTAGE OF AND USED IT TO
CONDUCT SOME RESEARCH.
WE ARE AWARE THAT A NUMBER OF
INSTITUTIONS INCLUDING LIBRARIES
ARE SETTING UP REPOSITORIES OR
ALREADY SETTING THEM UP AND ARE
GOOD.
THEY MEET INSTITUTIONAL NEEDS
AND AWARE OF THE NEEDS OF
GOVERNMENT AGENCIES LITHE NSF OR
NIH THAT HAVE DATA SHARING
REQUIREMENTS AND DEAL WITH
SECURITY.
PUB MED CENTRAL CAN BE USED AS A
REPOSITORY.
YOU PUBLISH A PAPER AND AS
REQUIRED THE PAPER IS POSTED IN
PUB MED CENTRAL.
IF THE DATA THAT GOES WITH THE
PAPER IS LESS THAN A CERTAIN
SIZE AND I DON'T REMEMBER WHAT,
BUT LESS THAN A CERTAIN SIZE,
YOU CAN POST THE DETE THERE.
THESE ARE NOT NIH REPOSITORIES
AND THESE ARE REPOS TRIES THAT
ARE WIDELY USED IN THE
COMMUNITY.
BUDGET IS A BIG ISSUE.
WE ASK THAT PEOPLE THINK HOW
MUCH IS IT GOING TO COST TO MEET
THE CONDITIONS OF A DATA
MANAGEMENT SHARING PLAN.
WE OUTLINED WHAT KIND OF COSTS
ARE REASONABLE AND CAN BE
CONNECTED TO DIRECT COSTS AND
THERE'S OTHER THAT CONTINUE TO
BE MORE LINKED WITH INDIRECT
COSTS.
I SHOULD ALSO MENTION THE
NATIONAL LIBRARY OF MEDICINE
COMMISSIONED A STUDY WITH THE
NATIONAL ACADEMIES TO LOOK AT
THE ISSUE OF HOW MUCH DOES IT
COST TO SHARE DATA.
THIS IS HOW IT GOES.
THE PLAN IS SUBMITTED WITH THE
INITIAL APPLICATION.
THERE ARE TWO PARTS.
THE PLAN ITSELF, WHICH I BELIEVE
IS SUPPOSED TO BE TWO PAGES LONG
OR LESS.
AND THEN THERE'S ALSO
JUSTIFICATION FOR THE BUDGET.
WITHIN THE BUDGET JUSTIFICATION
YOU'RE SUPPOSED TO TALK ABOUT
THE DATA MANAGEMENT AND SHARING
PLAN AND THERE'S SUPPOSED TO BE
AN ENTRY TO HOW MUCH MONEY YOU
THINK YOU'LL NEED TO IMPLEMENT
THE DATA MANAGEMENT AND SHARING
PLAN.
THE PLAN WILL THEN BE ASSESSED
FOR THOSE TO BE FUNDED BY OUR
STAFF AND NIH STAFF AND ANY TIME
ALONG THE WAY IT CAN BE REVISED
AND PROGRESS REPORT ONE, TWO,
THREE YEARS LATER YOU CAN REVISE
THE PLAN AND WOULD REQUIRE
DISCUSSION WITH NIH STAFF AND
ONCE APPROVED IT BECOMES PART OF
THE TERM AND CONDITION OF THE
AWARD LIKE ANY OTHER.
THE INFORMATION ABOUT PROGRESS
IN MEETING THE PLAN SHOULD BE
INCLUDED IN ANNUAL PROGRESS
REPORTS AND FAILURE TO COMPLY IS
A PROBLEM.
THAT MAY AND IS CONSIDERED LIKE
ABE OTHER COMPLIANCE VIOLATION.
BY THE WAY, WE'RE THINKING IN
THIS PARTICULAR CASE WE MAY
LEARN ABOUT FAILURES TO COMPLY
FROM OTHER PEOPLE IN THE
RESEARCH COMMUNITY WHO WILL SAY,
LOOK, I TRIED TO GET ACCESS TO
THIS PARTICULAR DATA AND I'M NOT
ABLE TO GET ACCESS OR GIVEN A
HARD TIME OR WHATEVER.
THAT MAY BE A CUE TO US THERE'S
POTENTIALLY A PROBLEM.
WE KNOW THERE'S BEEN INTEREST IN
THE COMMUNITY AND THERE'S A
PAPER PUBLISHED IN CROSS
COMPUTATIONAL BIOLOGY BUT THE
CONTENT OF THE PAPER IS
APPLICABLE TO ANYTHING NOT JUST
COMPUTATIONAL TECHNOLOGY AND
LOOKING AT THE DATA MANAGEMENT
SHARING PLAN AND THIS CAME FROM
NORTHWESTERN UNIVERSITY.
IT'S A NICE PAPER WE'VE BEEN
ENGAGED IN EXTENSIVE OUTREACH.
THERE'S BEEN DOZENS OF WEBINARS,
SEMINARS, AND THAT WILL
CONTINUE.
THIS IS AN OPPORTUNITY TO THANK
THREE COLLEAGUES IN PARTICULAR,
THE OFFICE OF SCIENCE POLICY AND
JULIA FROM OUR OFFICE AND
EXTRAMURAL RESEARCH AND IN OUR
OFFICE OF EXTRAMURAL RESEARCH.
THE THREE HAVE BEEN WORKING ON
THIS TIRELESSLY FOR YEARS.
I KNOW YOU'RE AWARE OF THE
OFFICE OF SCIENCE AND TECHNOLOGY
POLICY MEMORANDUM PUT OUT LAST
AUGUST.
THIS MEMORANDUM WAS ENTITLED
ENSURING FREE IMMEDIATE AND
EQUITABLE ACCESS TO FEDERALLY
FUNDED RESEARCH.
THE PART THAT HAS DRAWN THE MOST
AMOUNT OF ATTENTION HAS BEEN ON
PUBLIC ACCESS.
REFERRING TO AND TO ARTICLES
PUBLISHED AND SUPPORTED BY THE
FEDERAL GOVERNMENT AND THE IDEA
IS SOME VERSION OF THOSE
ARTICLES SHOULD BE PUBLICLY
ACCESSIBLE WITHOUT EMBARGO AT
THE TIME OR SHORTLY AFTER
PUBLICATION.
THERE ARE TWO PARTS.
ONE IS ON THE DIGITAL PERSISTENT
IDENTIFIER.
HAVING DIGITAL IDENTIFIERS
CONNECTED TO ASPECTS OF RESEARCH
AND IDENTIFIERS TO GRANTS AND
PUBLICATIONS AND DATA SETS AND
TO PEOPLE.
AN EXAMPLE MIGHT BE THE ORCHID
I.D.
SCIENTIFIC DATA RESULT FROM
FEDERALLY FUNDED RESEARCH SHOULD
BE MADE FREELY AVAILABLE AND
PUBLICLY ACCESSIBLE BY DEFAULT
AT THE TIME OF PUBLICATION AND
SHOULD BE SUGGEST TO FEDERAL
AGENCY GUIDELINE FOR RESEARCH
RESPONSIBILITIES REGARDING DATA
MANAGEMENT AND SHARING PLANS.
I STARTED BY TALKING ABOUT THE
FACT THAT DATA SHARING HAS BEEN
PROMOTED AT THE HIGHEST LEVELS
OF GOVERNMENT INCLUDING FROM THE
WHITE HOUSE AND CONGRESS AND
HERE WE ARE SURFING BACK TO THE
WHITE HOUSE AGAIN AND IF THIS
STATEMENT CAME OUT IN A MEMO
LAST AUGUST ESSENTIALLY MATCHES
OUR DATA MANAGEMENT AND SHARING
POLICY AND WE FEEL WE CAN CHECK
THE BOX WE'VE DONE WHAT HAS BEEN
ASKED.
THANK YOU VERY MUCH FOR YOUR
ATTENTION TODAY.
HAPPY TO HEAR YOUR COMMENTS AND
QUESTIONS AND I HAVE HERE A LIST
OF A VARIETY OF RESOURCES BUT
PERHAPS THE MOST IMPORTANT
IMPORTANT RESOURCE IS
SHARING.NIH.gov AND IF YOU
HAVEN'T LOOKED AT IT ENCOURAGE
YOU TO DO SO.
TALE YOUR FRIENDS.
THANK YOU.
>>IT'S EXCITING TO SEE HOW YOU
COMPLIED THE INFORMATION AND
RESOURCES.
YOU DON'T WANT TO LEAN TOO MUCH
ON SAMPLE PLANS BUT THINK IT
WILL HELP PEOPLE VISUALIZE WHAT
THEY'RE TRYING TO DO AND WOULD
BE APPROPRIATE.
>>AND WITH THE OFFICE OF
SCIENCE POLICY AND MIKE'S OFFICE
THE OFFICE OF EXTRAMURAL
ACTIVITIES AS WELL AS THE OFFICE
OF EXTRAMURAL RESEARCH BECAUSE
THIS DOES AFFECT OUR INTRAMURAL
RESEARCHERS.
WE PUT TOGETHER A GROUP AT NCATS
FROM EACH OFFICE AND DIVISION
AND PROGRAM TO SHARE EXPERIENCES
AND WE PUT TOGETHER EDUCATIONAL
PROGRAMS FOR OUR STAFF.
AND IT'S CONNECTED TO THE PEOPLE
AT NIH.
I ENCOURAGE YOU IF YOU HAVE
ISSUES RELATED TO OUR PROGRAMS,
WHICH ARE A LITTLE DIFFERENT AND
MANY NIH PROGRAMS WE'RE HERE TO
HELP.
THANKS.
>>LIVING IN THE RARE DISEASE
WORLD THIS IS SOMETHING WE THINK
ABOUT A LOT.
EVEN NOT JUST GENOMIC DATA BUT
CLINICAL DATA CAN BE HIGHLY
IDENTIFYING FOR SMALL GROUPS.
I ASSUME WE ALL HAVE GOOD
GUIDANCE ON THAT.
I'M MISSING THE SESSION ON
CHILDREN'S NATIONAL ON DATA
POLICY ON THIS.
I FIGURE I'M GOING STRAIGHT TO
THE SOURCE AND GETTING BETTER
DATA.
HOW DO YOU WAND TO HANDLE THE
SMALL GROUP SIZES?
>>THERE'S A DISCUSSION AND WE
HAVE HAD TO DEAL WITH THIS
ALREADY WITH SOME OF OUR GENOMIC
WORK.
AND I THINK PART OF THE REASON
WHY THE POLICY WAS SET UP THE
WAY IT WAS, WHICH IS THAT WE'RE
NOT SAYING YOU MUST SHARE ALL
DATA AND SHE VERY MUCH -- SHARE
ALL DATA AND THANK YOU VERY MUCH
AND HAVE A NICE DAY BUT YOU NEED
A SHARING PLAN YOU NEED TO
DESCRIBE.
THE REASON IS WE FUND HIGHLY
HERTERO GENUS -- HETEROGENEOUS
RESEARCH AND THAT'S THE REASON
WHY WE SET IT UP.
WE'RE ASKING FOR PLANS AND WE
LOOK AT WHAT'S FEASIBLE AND
REASONABLE AND POSSIBLE.
>>THANK YOU VERY MUCH FOR THAT.
>>IT'S INTERESTING WHEN IT HITS
THE IRB WITH OUR RARE DISEASE
PATIENTS.
>>PAUL.
QUESTION OR COMMENT?
>>TWO QUESTIONS.
>>WE'VE DONE WELL WITH OUR
PARTICULAR STUDY.
FUNDED HAS ENDED AND I'VE CHOSEN
THE WRONG REPOSITORY AND GOING
OUT OF BUSINESS OR JACKING UP
THE PRICE, ETCETERA.
WHAT'S THE RESPONSIBILITY OF THE
INSTITUTION TO KEEP THE DATA
SHARING?
>>YOU'RE DESCRIBING WHERE
THROUGH NO FAULT OF YOUR OWN
YOU'RE STUCK.
YOU'RE IN A DIFFICULT SPOT.
THE SECOND IS A CONCEPT AND
GRANT LAW REGARDING WHAT IT
MEANS WHEN A GRANT IS OVER.
>>THERE'S LOTS OF
RAMIFICATIONS.
ONCE A GRANT IS CLOSED, THAT'S
IT, THE GRANT IS CLOSED AND NO
MORE CAN BE SPENT ON GRANT FOR
THE PURPOSE OF THAT GRANT.
WHAT HAPPENS AFTER GRANT
CLOSEOUT IS VERY INTERESTING
QUESTION.
IN SOME CASES IT MAY BE
RELATIVELY EASY TO MANAGE.
FOR EXAMPLE, IF THE DATA ARE
DEPOSITED IN AN NIH REPOSITORY
IT'S WITH US AND IF WE HAVE A
PROBLEM WITH OUR REPOSITORY,
THAT'S AN ISSUE.
THAT WILL BE PROBABLY MORE
LONGER TERM.
ANOTHER POSSIBILITY IS YOU MAKE
A PURPOSE OF THREE YEARS WORTH
OF SHARING AND THAT PURCHASE IS
MADE DURING THE TIME THE GRANT
IS ACTIVE.
THAT'S WHEN THE MONEY GETS SPENT
AND WHAT YOU'RE DOING IS
BASICALLY BUYING LONGER TERM
SUBSCRIPTION THAT MAY VERY WELL
WORK.
THE QUESTION YOU'VE ASKED IS AN
IMPORTANT ONE.
FORTUNATELY, WE DO HAVE A FEW
YEARS BEFORE WE HAVE TO
IMMEDIATELY WORRY ABOUT IT.
AND WE ARE NOW IN THE PROCESS OF
GOING THROUGH WHAT WE AFFECT
AFFECTIONATELY CALL ARE UNIFORM
GUIDANCE THAT COVER GUIDANCE
ACROSS THE ENTIRE FEDERAL
GOVERNMENT.
WHAT I WAS TALKING WITH YOU
ABOUT WITH GRANT CLOSE OUT ARE
THE PRINCIPLES WITHIN THE
UNIFORM GUIDANCE.
THESE ARE SOME QUESTIONS WE'LL
BE THINKING ABOUT AS WE ENGAGE
WITH COLLEAGUES DOWNTOWN IN HOW
THE GRANT REGULATIONS ARE MOVED
FORWARD AND EFFECTIVELY THE
SCIENCE IS AHEAD OF THE LAW AND
WE HAVE TO FIGURE OUT HOW TO DO
THAT.
>>YOU CAN SHARE THINGS WELL OR
NOT SO WELL.
I WORRY THERE'S NOT ENOUGH
PRIVACY EXPERTS AT ALL THE
INSTITUTIONS TO SHARE VERY WELL.
YOU CAN IMAGINE A SCENARIO WHERE
THE HARVARDS OF THE WORLD GET
GOOD AND THE UNIVERSITY OF SOUTH
TENNESSEE IS NOT SO GOOD.
YOU'RE GRADING ON THE BASIS OF
THE QUALITY THAT CAN CREATE
FURTHER DISPARITIES.
>>THAT'S TRUE.
ONE OF THE CONCERNS WITH ANY OF
THE POLICIES INCLUDING DATA
SHARING AND PUBLIC ACCESS IS
MIGHT THAT ACTUALLY WORSEN
DISPARITIES BETWEEN RESOURCES.
THAT'S WHAT WE'LL PAY ATTENTION
TO.
I'LL SAY PART OF MY JOB IS TO
DEAL WITH COMPLIANCE ISSUES AND
I HAVE SEEN I'VE SEEN WELL
RESOURCED INSTITUTIONS HANDLE
COMPLIANCE WELL AND NOT SO WELL.
YOUR POINT IS SPOT ON BUT IT'S A
COMBINATION OF HOW RESOURCED AN
INSTITUTION IS AND THE CULTURE
WHAT CULTURE DOES THE
INSTITUTION HAVE.
>>I'M EXCITED TO SEE THE
SHARING OF DATA HAS BEEN A
CORNERSTONE FOR WHAT WE HAVE
BUILT AND HAVE BEEN ABLE TO
ACHIEVE.
ONE COMMENT YOU ADDRESSED IS THE
PLAYING FIELD IS NOT LEVEL.
AND WE HAVE PEOPLE DOING THE JOB
FOR A LIVING AND WE MADE AN
EFFORT AND ACTIVELY LEARNED WHAT
OUR USER COMMUNITY WANTS TO SEE
AND HOW TO SHARE FROM PATIENT
PARTICIPANTS UP TO PROFESSIONAL
BIO INFORMATITION -- BIOB
BIOINFORMATICIAN AND WE WANT
THEM TO STAY IN CLINICAL CARE.
IF YOU CONTINUE THE LISTENING
SESSION NOT ONLY IN BUILDING
POLICY BUT OVER THE NEXT THREE,
SIX, 12 MONTHS TO SEE HOW THE
POLICY IS IMPLEMENTED AND LEARN
FROM PEOPLE WHO ARE STRUGGLING
AND WATCH WHAT IS A SUBMISSION
STRUCTURE AND ARE THERE PARTS
WHERE YOU SEE LESS GRANTS COMING
IN SO WE DON'T RUN INTO
UNINTENDED CONSEQUENCES.
THE FINAL POINT SHARING DATA
EARLY AND OFTEN PAYS FOR
INVESTIGATORS.
THEY GAIN VISIBILITY AND WHAT IS
THE ADVANTAGE LIKE IN THE
EXAMPLE OF SHARING DATA EARLY
AND OFTEN AND IN THE FEDERAL
MANDATE.
>>EXCELLENT POINTS.
>>WE SEE THIS AS A LEARNING
EXPERIENCE.
WE'LL BE PAYING CLOSE ATTENTION
AND GOING ON YEARS.
ANG WHAT CAN WE DO TO MAKE THE
PROCESS MORE USER FRIENDLY AND
YOU MAKE A GOOD POINT.
ONE MESSAGE WE'D LIKE TO PROMOTE
AND MAYBE I DID A LITTLE BIT
HAVING HIGH QUALITY DATA
AVAILABLE OUT THERE CAN HELP
JUNIOR INVESTIGATORS GET
PROGRAMS GOING.
AND MY OWN EXPERIENCE AT THE
TIME I WAS OBTAINING SHARED DATA
FROM NHLBI I WAS BUILDING UP MY
OWN RESEARCH PROGRAM AND HAD NOT
REACHED THE LEVEL OF MATURATION.
I WAS ABLE TO DO SOMETHING AND
LEARN SOMETHING AND WHEN YOU SEE
AND WEN YOU SEE HOW THEY
ANNOTATE IT, CURATE IT, DESCRIBE
IT, IT'S A ROLE MODEL IF IT'S
BEING DONE WELL, I WANT TO DO
THE SAME THING.
I WANT TO MAKE MY DATA LOOK THIS
GOOD SO WHERE OTHER SCIENTISTS
MAY DISAGREE WITH MY
INTERPRETATIONS, THEY'LL
APPRECIATE WE'RE TRYING TO DO
CAREFUL WORK.
>>WE SAW DATA SET ON A SILVER
PLATE TO YOUNG INVESTIGATORS TO
ATTRACT THEM TO SCIENCE AND THIS
WHAT THIS WILL DO IF I AM --
IMPLEMENTED EFFECTIVELY.
>>I APPRECIATE THE CONVERSATION
AND EFFORT.
I SEE THIS THROUGH THE LENS OF
EVACUATE BEEN A PART OF
FRUSTRATION OF COMMUNITIES AND
THE LONG RUNWAYS AND TIME LINES
TO HAVE DATA PUBLISHED AND
SHARED AND COMMUNITIES THAT
ATTEMPTED TO FACILITATE
RELATIONSHIPS BETWEEN
INVESTIGATORS AND RUN INTO
BARRIERS BECAUSE THE DATA
SHARING BARRIERS WERE
INSURMOUNTABLE.
AND EN FORCES DATA SHARING
POLICIES AND WE RAN INTO THERE
WEREN'T TEETH IN THE EXISTING
POLICIES.
WE WERE PLEASED TO SEE
LEGISLATION NOW IMPLEMENTED ON
THE BACK END.
THANK YOU FOR THE WORK.
A COUPLE QUESTIONS AROUND THAT.
WHEN WE TALK ABOUT DATA AND I
KNOW IT'S MANY THINGS, MANY
FORMS.
A LARGE SHARE OF THE DATA IS
PATIENT EXPERIENCE.
AND OFTEN TIMES THE PATIENTS WHO
PARTICIPATED IN THESE STUDIES
AND CLINICAL TRIALS AND NATURE
HISTORY STUDIES AND I'M
WONDERING IF WHEN FORMULATING
THIS POLICY WERE THERE ANY
PATIENTS INVOLVED OR PATIENT
ADVOCACY ORGANIZATIONS OR
PATIENT GROUPS THAT HAVE BEEN
INVOLVED IN RESEARCH INVOLVED IN
POLICY MAKING AND CONSIDERATIONS
FOR THE POLICY?
SO THAT WOULD BE -- I'LL PUT MY
QUESTIONS OUT AND YOU CAN ANSWER
THEM.
WHEN YOU LISTED THE REASONS FOR
NOT SHARING DATA.
WHEN YOU WORK WITH INVESTIGATORS
TO DEVELOP A PLAN, IT JUST FELT
TO ME THE THREE OR FOUR BULLETED
REASONS THEY FELT BROAD AND
BIASANCED -- BIASSED
POPULATIONS AND MAYBE MORE
SPECIFICITY AROUND THAT AND
MAYBE SOMETHING TO WATCH FOR
THAT WE DON'T WANT TO DEVELOP
POLICIES BIASSED AGAINST
POPULATIONS OVER OTHERS BECAUSE
DATA SHARING IS AN OPERATOR --
PRIORITY FOR OUR COMMUNITIES AND
DON'T WANT TO NAD VINADVERTENTL
IDENTIFIED FOLKS BUT THE LAST IS
AROUND THE BARRIER WE RAN INTO
BEFORE IS THE QUESTION OF THE
TEETH.
I SAW YOU TALKED ABOUT THE LACK
OF FOLLOW THROUGH IMPACT FUTURE
FUNDING IS THE ABILITY FOR NIH
TO GO BACK IF SOMEBODY HASN'T
COMPLIED AND WOULD THIS IMPACT
THEIR EXISTING FUNDING?
COULD THIS IMPACT PUBLICATIONS?
ARE THERE ANY TEETH IN THIS NEW
POLICY IS THE QUESTION?
>>I'LL PLAY A LITTLE IGNORANCE
BECAUSE I WAS NOT AT THE TABLE
WHEN THAT WAS HAPPENING AND PART
OF THE PROCESS BUT NOT TO THE
SAME DEGREE AS MY COLLEAGUES
FROM THE OFFICE OF SCIENCE
POLICY WERE AND I BELIEVE THE
ANSWER TO YOUR QUESTION IS YES.
AND AS THE PROCESS EVOLVED WE
DON'T BANT TO EFFECTIVELY BIAS
CERTAIN POPULATIONS.
WE'LL GET TENS OF THOUSANDS OF
SHARING PLANS.
WE HAVE SOME EXPERIENCE BASED ON
THE POLICIES THAT ALREADY EXIST
BUT MUCH OF THIS HAS BEEN
THEORETICAL OR HYPOTHETICAL.
NOW IT WILL BE REAL.
ONE THING WE'LL DO IS WE'RE
GOING DEVELOP A PANEL OF NIH
STAFF WHO HAVE STRONG INTEREST
IN EXPERTISE IN DATA MANAGEMENT
SHARING TO HELP US DEAL WITH THE
INTERESTING ONES PLANS THAT ARE
CHALLENGING FOR A VARIETY OF
REASONS.
THE OTHER IS TO GATHER
BASICAL
BASICALLIAL -- BASICALLY A
KNOWLEDGE BASE AND WHERE THERE
ARE PROBLEMS TO HELP THINK THIS
THROUGH GOING FORWARD.
AND WE GIVE GRANTS TO
INSTITUTIONS NOT INDIVIDUAL
SCIENTISTS.
AND WHEN THERE ARE COMPLIANCE
CONCERNS WE CAN TAKE A VARIETY
OF ACTIONS.
WE CAN TAKE ACTIONS ON SPECIFIC
GRANTS AND WE CAN SUSPEND A
GRANT OR REQUEST OR REQUIRE
THERE'S NEGOTIATION OF TERMS AND
CONDITIONS.
WE CAN REQUEST RETURN OF FUNDS.
AND WE CAN ISSUE NEW FUNDING ON
CERTAIN TYPES OF PROJECTS.
WE HAVE A WIDE VARIETY OF LEVERS
FOR US THAT AND WE USE THESE
JUDICIALLY AND CAREFULLY WHEN
DEALING WITH COMPLIANCE
CONCERNS.
UP TO NOW WE HAVEN'T HAD TEETH
EXCEPT FOR SPECIFIC POLICIES
LIKE PROJECTS LIKE GENOMIC DATA
SHARING.
NOW, BECAUSE WE'RE ESSENTIALLY
SAYING TO GRANT RECIPIENT, YOU
NEED TO FOLLOW YOUR OWN PLAN.
IT'S LIKE CONFLICT OF INTEREST.
AND YOU HAVE TO FOLLOW YOUR OWN
PLAN AND IF YOU DON'T THEN
YOU'RE OUT OF COMPLIANCE AND
THEN WE HAVE A VARIETY OF LEVERS
WE CAN USE AND WE DO USE THEM IN
AN APPROPRIATE WAY.
THANK YOU.
>>TWO THINGS.
I SIT ON THE BLACK WOMEN HEALTH
IMPERATIVE.
THOSE ARE TWO GROUPS YOU SHOULD
TALK TO TO SEE HOW THEY IMPACT
ENROLLING IN STUDIES THERE'S
ALREADY HUGE TRUST ISSUES TO
BEGIN WITH AND YOU SAY YOUR DATA
COULD BE SHARED OR GOING TO BE
SHARED.
I WOULD TRY TO GET INPUT IN DATA
GOING FORWARD.
AND ANOTHER INTERESTING QUESTION
ABOUT TEETH AND ENFORCEMENT.
HISTORICALLY ONE OF THE HARDEST
PLACES TO GET MATERIALS AND DATA
IS FROM WHERE?
THE NIH?
HOW ARE YOU GOING ENFORCE THIS
INTERNALLY AND MAKE FOLKS DO
THIS.
>>AND THERE'S SPECIFIC GRANTS
BUT JUST AS THE POLICY TOOK
EFFECT YESTERDAY FOR EXTRAMURAL
PROJECTS THEY APPLY TO
INTRAMURAL PROJECTS.
>>WHERE ARE THE TEETH?
>>I'M THE EXTRAMURAL PERSON
SO --
>>AND I CAN SEE THIS PART OF
BSC REVIEW AND BOARD OF
SCIENTIFIC COUNSELORS.
IF YOU'RE NOT SHARING DATA,
YOU'RE NOT BEING AN OUTSTANDING
SCIENTIST.
>>AND LET FOLKS KNOW INTERNALS
ARE NOT AND IT'S SURPRISING TO
ME.
>>MIKE, I FOUND YOUR
PRESENTATION REALLY HELPFUL.
VERY TIMELY AS WELL.
AND THERE'S REPOSITORIES
AVAILABLE TO THE PUBLIC AND I
DIDN'T SEE AND THAT'S WHY MY
QUESTION ALL OF US RESEARCH
PROGRAM INCLUDED AND WHAT
TRIGGERED THE QUESTION IS
LOOKING AT THE CONSORTIUM OF
PROGRAMS AND WE ARE USING THE
ALL OF US MASTER DATA IS TO
RECRUIT WORK BENCH RESEARCHERS
AT OUR INSTITUTION AND THE IDEA
IS TO ALSO RECRUIT EVEN
COMMUNITY MEMBERS.
STUDENTS, TO POTENTIALLY USE THE
DATA SO THE REACH IS VERY
INTERESTING.
AND NOT ONLY THAT.
PART OF THE COMMITMENT IS TO USE
COMMUNITY HEALTH WORKERS TO
CONTINUE COLLECTING MORE DATA
AND THIS IS BEING PAID BY NIH.
I THINK THAT SPECIFICALLY NCATS,
IF I'M NOT MISTAKEN.
IT'S A MODEL OF USING REPOSITORY
DATABASES THAT TIE AND TAKES
MILLIONS OF DOLLARS.
IF YOU CAN COMMENT I'D
APPRECIATE IT.
>>THE "ALL OF US" PROGRAM IS A
GREAT EXAMPLE.
AND WE LOOK AT THE CONCEPTION OF
THE "ALL OF US" PROGRAM FROM THE
BEGINNING.
AND THE IDEA DATA WOULD BE
SHARED WAS THAT WAS CORE TO TO
THE PROJECT FROM THE ORIGINS.
I THINK IT'S GREAT EXAMPLE.
I WASN'T THINKING OF IT AS A
REPOSITORY IN THE SENSE OF
WRITING AN RO1 GRANT FROM AN
UNIVERSITY SOMEWHERE I WOULDN'T
PUT MY DATA INTO ALL OF US
BECAUSE IT'S NOT PART OF THE ALL
OF US PROGRAM BUT FOR FUTURE
VERSIONS OF THIS TALK I WILL
INCLUDE ALL OF US AS AN EXAMPLE
OF A DATA SHARING RESOURCE WE
HOPE THE COMMUNITY WILL TAKE
ADVANTAGE OF AND AS AN EXAMPLE
OF HOW DATA SHARING CAN BE
POWERFUL.
>>THANK YOU.
>>AND FROM ABOUT WHAT I GATHER
THE DATABASE HASN'T BEEN USED
WIDELY AND GETTING ARTICLES
PUBLISHEDED.
AND THERE'S AN EMPHASIS ON
RECRUITING EARLY CAREER
RESEARCHERS FROM DIVERSE
BACKGROUNDS TO HELP WITH THEIR
CAREER DEVELOPMENT WHICH I THINK
IS GREAT THE NIH IS INVESTING IN
THOSE EFFORTS.
>>THANK YOU.
>>THANK YOU VERY MUCH, MIKE.
WE APPRECIATE IT.
>>THANK YOU, CLAIRE AND ANNA
AND JONI AND HAVE A GREAT
AFTERNOON.
ENJOY THE REST OF THE MEETING.
>>THANKS, MIKE.
>>LOOKS LIKE JONI'S BACK.
BACK OVER TO HER.
>>THANK YOU, MIKE.
I APPRECIATE THAT AND YOUR
LEADERSHIP AT THE NIH ON THESE
ISSUES AMONGST THE OTHER THINGS
YOU DO.
I APPRECIATE IT.
NEXT WE HAVE A PRESENTATION ON
FROM THE OFFICE OF SPECIAL
INITIATIVES WITH DR. DAN TAGLE.
I'LL LET DAN GO AHEAD AND TAKE
OVER.
DAN.
>>THANK YOU, JONI.
GOOD AFTERNOON, EVERYONE.
THANK YOU FOR HANGING IN THERE.
THIS LOOKS LIKE WE'RE IN THE
HOME STRETCH AND THIS WILL BE
THE LAST PRESENTATION.
I'M GOING GIVE A PROGRAM UPDATE
BUT FROM MY OFFICE AND PRIMARILY
ONE PROGRAM WITHIN THE OFFICE OF
SPECIAL INITIATIVES.
SO NEXT SLIDE, PLEASE.
JUST TO GIVE YOU AN IDEA WHERE
WE FIT.
THE OFFICE OF SPECIAL
INITIATIVES IS ONE OF SIX
OFFICES WITHIN THE NCATS
ORGANIZATION.
WE'RE MADE OF A DIVERSE GROUP OF
SCIENTISTS STARTING WITH PROGRAM
OFFICERS COMING FROM DIFFERENT
BACKGROUNDS OF CHEMISTRY AND
MICROBIOL
MICROBIOLOGISTS, BIOFPHYSICISTS
AND BIOMEDICAL ENGINEERS AND WE
HAVE PRESENTERS TODAY WHO ARE
PROGRAM OFFICERS SOME JOINED TWO
MONTHS AGO AND CAME FROM AND GOT
A Ph.D. IN CHEMISTRY FROM THE
UNIVERSITY OF WISCONSIN AND
JOINED FROM NIGMS.
THE OTHER HAND ARE ABLE
ASSISTANTS IN TERMS OF THE
PROGRAM MANAGERS AND A FELLOW
FROM NCI AND IS A BIOMEDICAL
ENGINEER BY TRAINING.
TALENTED GROUP OF PEOPLE AND
DIVERSE BACKGROUND.
THIS IS WHAT WE ARE DOING AT THE
OFFICE OF SPECIAL INITIATIVE.
WHAT WE DO IS TO ADDRESS
TRANSLATIONAL PROBLEMS THROUGH
INNOVATIVE SOLUTIONS BY
DEVELOPING AND IMPLEMENTING
DISRUPTIVE TECHNOLOGIES USING
INTERDISCIPLINARY APPROACHES IN
PUBLIC-PRIVATE PARTNERSHIP.
WHAT I DIDN'T HAVE ARE SLIDES
WITH OUR PARTNERS AND PATIENT
ADVOCACY GROUPS AND OTHER
GOVERNMENT AGENCIES SUCH AS
NASA, DARPA, BARDA, THE V.A.,
NSF.
THE OUTCOME OF THE PROGRAMS ARE
INTENDED TO BE CATALYTIC AND
TRANSFORMATIVE THAT RESULTS
TYPICALLY IN A PARADIGM SHIFT IN
THE FIELD.
SHOWN HERE ARE A LIST OF ALL THE
DIFFERENT PROGRAMS WE HAVE BEEN
ENGAGED IN AT THE LAST PROGRAM
UPDATE A YEAR AGO I SPOKE ABOUT
TISH CHIPS OR THE SYSTEMS FOR
DRUG SCREENING.
TODAY AND THIS AFTERNOON WE'LL
TALK ABOUT A SPECIALIZED
PLATFORM FOR AUTOMATED
CHEMISTRY.
THIS IS PRIMARILY GOING WITH THE
TAGLINE OF MORE TREATMENT TO ALL
PEOPLE BY EMPLOYING ANIMATION
AND ARTIFICIAL INTELLIGENCE HOW
TO DIVERSIFY OUR LIBRARIES AND
CHEMICAL COMPOUNDS WE'RE DOING.
AND LOOK AT THE EXTRAMURAL AND
INTRAMURAL PART OF NCATS AND ONE
WILL BE GIVEN BY DR. RUDNICKI
AND THE OFFICER FROM THE
PRECLINICAL INNOVATION WILL GIVE
THE INFRASTRUCTURE AND
ORGANIZATION THAT WE ARE
BUILDING CAPACITY WITHIN OUR
INTRAMURAL PROGRAM SO EXTRAMURAL
SCIENTISTS CAN GAIN ACCESS TO
THE RESOURCES.
WITH THAT OVER TO DR. RUDNICKI
TO TAKE OVER.
>>I'M LEADING THE EXTRAMURAL
SIDE OF THE PROGRAM.
THIS IS TO HAVE INTRAMURAL AND
EXTRAMURAL PROGRAMMING AND I'LL
START WITH UNMET NEED CENTRAL TO
THE DEVELOPMENT OF THE PROGRAMS.
WE THINK OF ALL CHEMICAL
MOLECULES.
THE SPACE IS HUGE, 10 TO THE 60
OF MOLECULES.
IN COMPARISON THE BIOLOGICAL
SPACE WHICH RESPONDS TO THE
MOLECULES WE WISH TO MODIFY
DISEASES THE SPACE IS SMALLER.
IT'S EASY TO MAP THE HUGE
CHEMICAL SPACE TO LARGER
BIOLOGICAL SPACE AND ONE OF THE
FOUR TRANSLATIONAL CHALLENGES.
THE REASON IS THE CHEMICAL TOOLS
WE HAVE TO EXPLORE CHEMICAL
SPACE IS NARROW.
IN ADDITION TO THE CHEMICAL
TOOLBOX WE'RE NOT VERY GOOD AT
PREDICTING CHEMICAL OR ACTIONS
AND WE HAVE TARGETS AND PRODUCE
CHEMICALS THAT ACHIEVE OR REDUCE
CHEMICALS AND STRUCTURES CHEAPLY
ENOUGH.
IT TAKES 10 TO 15 YEARS TO BRING
THE DRUG TO THE MARKET AND OVER
$2.6 BILLION.
IF WE COULD SHORTEN THIS WE
SHOULD BE ABLE TO ACCELERATE
DRUG DISCOVERY AND DEVELOPMENT.
HOW CAN WE DO THIS?
AND HAD A QUESTION, WHICH AREA
SHOULD NCATS FOCUS ON TO
ACCELERATE TRANSLATION.
AND THERE WAS EXPERIENCE IN
USING AUTOMATION IN BIOLOGY AND
LOOKING AT CHEMISTRY, DARPA HAD
ONE PROBLEM FOCUSSING ON
AUTOMATION AND CHEMISTRY.
NIGMS HAD A PROBLEM IN RELATION
TO GLYCOSCIENCE AND INDUSTRY HAD
EFFORTS FOCUSSING ON AUTOMATION
OF DIFFERENT CHEMICAL PROCESSES.
THE COMMON DENOMINATOR HERE WAS
NONE OF THESE EFFORTS WERE
FOCUSSING ON THE NEW
CHEMISTRIES.
THIS WAS ALL ABOUT KNOWN
CHEMISTRIES.
WE WERE INTERESTED IN
DISCOVERING NEW CHEMICAL SPACE.
WE ORGANIZED THE WORKSHOP AND
WERE INVITED BY A SPECTRUM OF
STAKEHOLDERS WITH EXPERTISE IN
CHEMISTRY IN AUTOMATION AND A.I.
AND MACHINE LEARNING AND ASKED
THEM WHAT MAIN ROAD BLOCKS ARE
TO AUTOMATING CHEMISTRY AND
WHERE THE LOW HANGING FRUITS
ARE.
THIS IS TO USE HOW AUTOMATION
HAS IMPACTED HIGH THROUGHPUT
SCREENING AND TISSUE CULTURE AT
NCATS.
IF YOU CARE THIS TO HOW
CHEMISTRY IS DONE, THIS LOOK
DIFFERENT AND IT DOESN'T REALLY
LOOK MUCH DIFFERENT FROM HOW IT
WAS DONE A HUNDRED YEARS AGO.
THE CULTURAL ROAD BLOCK IS
CHEMISTRIES WERE CRAFT LIKE
SCIENCE AND CHEMISTS CAN DEVELOP
BEAUTIFUL MOLECULES AND
FREQUENTLY THE CHOICE TO GO AND
DEVELOP INTUITION AND OF COURSE
THAT'S NOT SOMETHING YOU CAN
AUTOMATE EASILY.
IT'S LOGICAL THE FIELD WOULD
HAVE RESERVATION TOWARDS
AUTOMATION.
AND WE LOOKED AT HOW CHEMISTS
COLLECT DATA AND FOUND OUT IF
YOU WANT TO GO FROM THE IMAGE TO
THE LEFT -- SOMETHING THAT LOOKS
MORE LIKE THIS, WHICH IS MORE
APPROPRIATE FOR AUTOMATION, WE
REALLY NEED TO CHANGE THE WAY WE
CAPTURE DATA.
WE NEED TO HAVE MORE VALIDATION
AND INCREASE THE VISIBILITY AND
NEEDS TO BE ACCESSIBLE SO A WIDE
RANGE OF CHEMISTS CAN MINE
THROUGH IT.
WE NEED TO MAKE SURE THE VIDEO
OF THE DATA IS NOT BIASSED AND
STANDARDIZED AND NEED TO BE ABLE
TO ADD ADDITIONAL DATA POINTS
AND NEED CHEMICAL STRUCTURES
COMPATIBLE WITH MACHINE LEARNING
AND THE BEST DATA IS CAPTURED IN
REAL TIME.
THE DATA SHOULD BE AVAILABLE FOR
RESEARCHERS TO USE IT.
AUTOMATED CHEMISTRY WILL
LIBERATE CHEMISTS FROM MUNDANE
WORK AND BE ABLE TO SPEND MORE
TIME ON DIFFICULT SYNTHETIC
CHALLENGES AND INTELLECTUAL
PURSUITS.
ONCE WE HAVE INCREASED DATA
SHARING THIS WILL REDUCE
DUPLICATION AND ALLOW US TO
ADOPT NOVEL NOVELS IN
LABORATORIES MORE QUICKLY.
AND ONCE WE MINIMIZE HUMAN ERROR
IT SHOULD BE REPRODUCIBLE.
WHO WOULD BE THE DESIRED USERS?
EXPERT CHEMISTS TO DEDICATE MORE
TIME TO COMPLEX TASKS AND THEY
CAN INCREASE THEIR PRODUCTIVITY.
WHEN YOU APPLY AUTOMATION YOU
WANT TO DEVELOP A SYSTEM USED BY
MANY INCLUDING BIOLOGISTS AND
INFORMATICISTS AND ANYONE ABLE
TO FORMULATE A MOLECULAR
HYPOTHESIS.
WHAT WOULD BE THE OPTIMAL
OPERATION MODEL?
WE CAME THE CONCLUSION THE BEST
WOULD BE HAVE CAPITAL INTENSIVE
ROBOTIC AUTOMATION SITES AND TO
SUPPORT DEVELOPMENT OF
AFFORDABLE DEVICES BECAUSE NOT
EVERY CHEMICAL LAB CAN ADAPT TO
AUTOMATION BUT BE ABLE TO
AUTOMATE CERTAIN PROCESSES WOULD
BE HELPFUL.
AND WHAT WE HAVE LEARNS WE THEN
DECIDED TO PROPOSE DEVELOPMENT
OF PROGRAMS AS SPIRE AND THE
GOAL IS TO ADDRESS THE ROAD
BLOCKED BY COMBINING AUTOMATION,
ENGINEERING AND CHEMISTRY AND
MACHINE LEARNING AND BIOLOGICAL
SCREENING AND IN A NUTSHELL OUR
VISION WAS TO BE ABLE TO PREDICT
NOVEL STRUCTURES CAPABLE OF
MODULATING SPECIFIC TARGET OF
FEATURES AND SYNTHESIZE THE
STRUCTURES, TEST THEM IN
BIOLOGICAL ASSAYS AND ALL THE
DATA FEEDBACK IN THE SYSTEM SO
WE CAN REFINE THE PROCESS.
LONG THE WAY TO HAVE DISCOVERY
AND DEVELOPMENT AND GOING FROM
EMPIRICAL TO PREDICTIVE SCIENCE.
IF YOU WANT TO READ MORE ABOUT
EVERYTHING I RED SO FAR MUCH IS
PUBLISHED IN OUR NATURE REVIEW
DRUG DISCOVERY PAPER I HAVE
INDICATED HERE.
THERE'S EXTRAMURAL PROGRAMMING
AND EXTRAMURAL INFRASTRUCTURE,
COMPETENCE IS DESIGN TO BRING
NOVEL, SAFE AND EFFECTIVE
TREATMENTS TO ALL PATIENTS MORE
QUICKLY AND AT LOWER COSTS.
IT BUILDS ON EMERGING
TECHNOLOGIES, PROMOTES
MULTI-DISCIPLINARY
COLLABORATION.
WE WANT TO BE ABLE TO ENGAGE IN
THE WIDE SPECTRUM OF
STAKEHOLDERS AND THIS IS GOING
TO BE CRITICAL AND SHOWS THE
POWER THE TREMENDOUS POWER OF
ASPIRE IN BEING ABLE TO ADDRESS
HEALTH DISPARITIES AND ADVANCE
HEALTH EQUITY.
IT AIMS TO DELIVER ON THE NIH
MISSION TO PRODUCE
REPRODUCIBILITY AND BRIEFLY
GOING OVER THE PROOF OF CONCEPT.
IN 2018, NIH LAUNCHED THE
INITIATIVE CALLED HELPING TO END
ADDICTION LONG-TERM.
SO HEAL IS AN INCREDIBLE PUBLIC
HEALTH CHALLENGE AND THE GOAL IS
TO IMPROVE PREVENTION AND
TREATMENT STRATEGIES FOR OPIOID
USE DISORDER AND ADDICTION.
AND NCATS ASPIRE WAS ENVISIONED
AS BEING ABLE TO TACKLE SUCH
HEALTH EMERGENCIES.
WE OBTAINED HEAL FUNDING AND USE
IT FOR PROOF OF PRINCIPLE BY
COMBINING SYNTHETIC CHEMISTRY
AND A.I. LEARNING WE CAN
DISCOVER NEW TREATMENTS FOR
PAIN, OPIOID USE DISORDER.
WE DESIGN A SET OF CHALLENGES
AND THE REASON WE SELECTED
CHALLENGES AS A FUNDING
MECHANISM IS THE MECHANISM
SUPPORTS EXECUTION OF THE MOST
INNOVATIVE IDEAS AT THE PHASE
APPROPRIATE TO ADDRESS HEALTH
EMERGENCY.
AND PEOPLE FROM ALL WALKS OF
LIFE CAN APPLY AND NOT ONLY FOR
THOSE SCIENTISTS THAT ARE
TYPICAL NIH APPLICANTS.
THE FIVE CHALLENGES WERE DESIGN
AND THEY WERE DESIGN TO ADDRESS
DIFFERENT AREAS IMPORTANT TO BE
DEVELOPED TO TACKLE THE BIG
HEALTH CRISIS.
THE FIRST WAS TO DEVELOP A
DATABASE TO CONTAIN THE
CHEMICAL, BIOLOGICAL AND
CLINICAL DATA RELEVANT TO PAIN
AND OVERDOSE AND THE DESIGN OF
NOTEBOOK THAT'S GOING TO BE ABLE
TO COLLECT ALL THE MOLECULES
RELEVANT TO THE AREA AND THE
THIRD CHALLENGE WAS TO DEVELOP
AND READ THE TWO DATABASES AND
MINE THE DATA AND THE FOURTH WAS
TO DESIGN BIOLOGICAL ASSAYS THAT
ARE GOING TO BE RELEVANT TO PAIN
AND OVERDOSE.
IF ANYONE HAS AN IDEA HOW TO
START PUTTING THE DIFFERENT
MODULES TOGETHER TO PROPOSE IT.
AFTER WE ANNOUNCED THE WINNERS,
THIS WAS A DESIGN STAGE AND WE
WERE INTERESTED IN THE NEXT
STAGE ON THE PROTOTYPE
DEVELOPMENT.
WE INVITED ALL THE WINNERS TO
COME TO NIH AND INTERACT WITH
EACH OTHER AND MAYBE FORM NEW
TEAMS SO -- AND WHEN WE HAD
REDUCTION TO CHALLENGE WE HAD
TALENTED INNOVATORS TASKED TO
PUT THE MODELS TOGETHER IN A
WORKING PROTOTYPE.
LAST OCTOBER WE ANNOUNCED OUR
GRAND PRIZE WINNER.
AND THEY HAVE HIGH THROUGHPUT
SYNTHESIS WITH A MACROSCOPY
APPROACH AND THE RUNNER-UP TEAM
WAS FROM A COMPANY AND TOGETHER
WITH THEIR COLLABORATORS FROM
THE UNIVERSITY OF GLASGOW AND
ARIZONA STATE UNIVERSITY THEY
DESIGN AN AUTOMATED CHEMICAL
SYNTHESIS MODULE FOR AN ASSAY
BASED MODEL.
THESE HAVE VALIDATED THE
PLATFORMS FURTHER TESTING THEIR
MOLECULES IN BIOLOGICAL ASSAYS.
AND THE NCATS TEAM AND
LABORATORY BEGAN TO ESTABLISH A
PLATFORM OF PHYSICAL AND VIRTUAL
MODULES FOR SYNTHETIC CHEMISTRY
AND ENGINEERING AND INFORMATICS
AND BIOLOGICAL TESTING.
THIS IS NOT THE POINT WHERE WE
FOCUSSED ON A SPECIFIC AREA OF
PAIN AND WANTED TO EXPAND THE
PROGRAM TO BE ABLE TO ADDRESS
ANY TRANSLATIONAL CHALLENGES.
WE DEVELOPED COLLABORATIVE
RESEARCH PROGRAM WHERE THE
INVITED RESEARCHERS TO TEAM UP
WITH OUR INTRAMURAL TEAM AND
HAVE THEN DEVELOP VIA PLATFORM.
WE DEVELOPED TWO INITIATIVES.
THE FIRST WAS FOCUSSING ON THE
PHYSICAL PLATFORM OR MODULE THAT
WILL BE INCORPORATED IN THE
ASPIRE PLATFORM.
THE SECOND FOCUSSED ON THE
VIRTUAL MODELS INCORPORATED IN
THE ASPIRE PLATFORM.
IT'S IMPORTANT TO REMEMBER THIS
IS TARGETED.
WE OPENED THE FIELD TO ANY
TRANSLATIONAL PROBLEM.
I THINK I WILL STOP HERE AND
PASS IT TO MICHAEL TO GIVE YOU
MORE UPDATES ON THE INTRAMURAL
SIDE OF THE PROGRAM AND HOW OUR
ORDs ARE HELPING OUR AS SPIRE
TEAM TO BUILD THE PLATFORM.
>>THESE DONE A WONDERFUL JOB
EXPLAINING IT'S A COLLABORATIVE
EFFORT AND MAKE NCATS SOMEWHAT
UNIQUE WE HAVE WHAT I LIKE TO
CALL AN INSIDE-OUT APPROACH AND
HAVE INTRAMURAL INVOLVEMENT BUT
CAN'T DO THESE THINGS WITHOUT
EXTRAMURAL AWARDEES AND IT'S
IMPORTANT TO TALK ABOUT DATA
SHARING AND ACCESS HOW TO
PROVIDE AN ENVIRONMENT CONDUCIVE
TO SCIENTIFIC RESEARCH AT SCALE.
>>ON ASPIRE IT'S TO REIMAGINE
THE ENTIRE ECO SYSTEM.
YOU HAVE INTEGRATED AUTOMATED
SOLUTION.
WE'RE LOOKING AT WHAT TYPICAL
AUTOMATED BENCHES LOOK LIKE NOW
IN LABORATORIES AND ON THE RIGHT
IS WHERE THE ORIGINAL SYNTHESIS
LABORATORY WE WORKED WITH AND ON
TOP A COMPANY AND THEIR FULLY
INTEGRATED ECO SYSTEM.
IT'S NOT ONE OR THE OTHER WE
NEED BOTH.
WE NEEDED AUTO MISED TEMPLATE
AND WE MENTIONED IN REGARD TO
CHEMISTS AND WE DON'T WANT TO
INFLICT AUTOMATION UPON THE
CHEMISTS.
WE WANT TO MAKE SURE THEY
EMBRACE IT BECAUSE IF THEY DON'T
EMBRACE IT WE HAVE A LIMITED
CHANCE OF SUCCESS.
THE OVER ALL GOAL IS TO DEVELOP
NEW THERAPIES THROUGH EXECUTION
AND CHEMICAL SYNTHESIS BY
GREATLY INCREASING ITERATION
TIME AND ANALYZING PARADIGM.
AS THE BECOME POPULAR TO
DISCUSS.
IT'S A NICE PAPER THAT CAME OUT
NOW IN 2019.
AND REALLY I THINK TO THE POINT
MADE IT PLAYS WELL TO NCATS'
STRENGTH.
WE'VE GOTTEN GOOD ON BIOLOGICAL
SCREENING AND HAVE GIVEN
PREVIOUS PRESENTATIONS AND HAVE
MED CAM INVOLVEMENT AND EFFORT
TO DO PROPER ANALYSIS.
HOW DO WE COMBINE THIS INTO A
CONTINUOUS PROCESS AND PLAY TO
THE STRENGTHS AND RECOGNIZE
THERE ARE GAPS IN PARTICULAR ON
THE AUTOMATION OF CHEMISTRY
ITSELF.
TO GET THIS DONE WE NEED TO
CREATE FROM THE GROUND FLOOR A
SMART LAB.
IT BEGS THE QUESTION FOR THIS
AND THE IDEA WE'RE TALKING ABOUT
AND NONE OF THE IDEAS ARE
RELATIVELY NOVEL WHAT'S MISSING
IS THE IMPLEMENTATION.
TO MAKE THE IDEA OF A SMART LOB
POSSIBLE WE NEEDED RADICAL
IMPROVEMENTS AND REAGENT
HANDLING AND FEEDBACK
TECHNOLOGIES AND ISOLATION
CAPABILITIES AND NEED PROCESSES
IN REGARD TO CHEMICAL REACTIONS
AND AUTOMATIC CHARACTERIZATIONS.
IN SOME CASES THEY'RE IN THE
WEEDS ELEMENT BUT TO AUTOMATE
THE DAY IN, DAY OUT PROCESSES A
CHEMIST WILL DO THAT WILL LEAD
US TO A CHANCE OF SUCCESS.
WHAT WILL THAT LOOK LIKE FROM A
PRACTICAL PERSPECTIVE FROM THE
APPROACHES NCATS ARE TAKING.
WE'RE LOOKING FOR A BENCH TOP
EXPLORER AND LOOK AT SAMPLES
FROM THE DEVELOPMENT OF NEW
CHEMISTRY AND TRACKING TO
PRODUCE THIS TESTABLE
HYPOTHESIS.
IN CONVERT WITH AN ANALYTICS
PLATFORM AND THIS IS A KEY
COMPONENT AND MOST CHEMISTRY
THESE DAYS STARTS IN A VIRTUAL
ENVIRONMENT SO WE WANT TO BRING
AND COMBINED THE PHYSICAL
CAPABILITIES OF THE BENCH WITH A
VIRTUAL ENVIRONMENT TO PLAN AND
ANALYZE RESULTS AND IT RATE OVER
TIME.
NCATS PUT A LOT OF RESOURCES
INTO THE DEVELOPMENT OF THE
COMPUTATIONAL PLATFORM AICP.
ONE IS OUR PHYSICAL LAB SPACE
WASN'T READY AND FROM A
COLLABORATIVE ASPECT AND FOLKS
REFERRING TO ALL OF US AND N3C,
THEY'RE AREAS WE'VE SHOWN THE
POWER OF COLLABORATIVE
ENVIRONMENT ENGAGE IN SCIENCE
AND BUILD OUT THE CAPABILITY TO
HAVE A COLLABORATIVE ENVIRONMENT
FOR RESEARCHERS TO WORK IN.
WE THINK ABOUT THE FRAMEWORK
WE'RE TALKING ABOUT REFERENCED
AND FROM THE EXTRAMURAL SIDE
FIRST WE WANT TO AUTOMATE THE
BENCH.
WE HAVE THE DASHBOARD AND USING
THE INTERNET OF THINGS AND
ARCHITECTURE TO TAKE LAB
EQUIPMENT AND CAPTURE
INFORMATION FROM LAB EQUIPMENT
THE CHEMISTS ARE USING AND IN
PARTICULAR FOLKS IN CHEMISTRY
AUTOMATION TO HAVE THAT IN AN
INFRASTRUCTURE AND THEN WE ALSO
HAVE THE LABORATORY ITSELF.
WE'RE SHOWING IN THE TOP RIGHT A
RENDERING WHEN WE FIRST GOT THE
PLANS FOR OUR C1 LAB SPACE AND
REFERS TO OUR 9800 BUILDING
HOPEFULLY WHEN COUNCIL'S IN
PERSON WE'D LOVE TO GIVE THEM A
TOUR AND WE HAVE PLANNED MODULES
FOR DIFFERENT ELEMENTS OF THE
ASPIRE PROCESS AND THE FRAMEWORK
IF YOU CAN ADVANCE ONE SLIDE, WE
NEED BOTH OF THESE CONNECTED.
THIS IS WHERE THE ASPIRE
COMPUTATIONAL PLATFORM IS
CRITICAL.
IT'S GOING TO BE WHAT LINKS NOT
JUST FOR NCATS RESEARCHERS AND
EXTERNAL RESEARCHERS THE ABILITY
TO INITIATE EXPERIMENTS
VIRTUALLY THAT EXECUTE
PHYSICALLY AND GET THE RESULTS
AND RINSE REPEAT UNTIL HOPEFULLY
LEADING TO NEW THERAPEUTIC
DISCOVERY.
THERE'S WORK TO DO TO GET THERE
WE STARTED ON.
WE'RE LOOKING TO HAVE THE
INSILICO DESIGN AND PREP IT,
ISOLATE IT, PURIFY IT AND THIS
IS WHAT WE DRUG DISCOVERY DMTA
CYCLE AND THE INDIVIDUAL STEPS
THAT HAVE TO TAKE PLACE AND
IDEALLY BE AUTOMATED OVER TIME.
WHERE WE'RE AHEAD OF NCATS AND
HAVE A LEG UP IS ON THE
BIOLOGICAL TEST.
WE'VE DONE EXTENSIVE AUTOMATION
GOING ON CLOSE TO TWO DECADES.
IT PLAYS TO OUR STRENGTHS.
WE'RE INVOLVED IN EVERY STEP 1-4
FROM THE DESIGN PREP AND
ISOLATION IN A MANUAL SPACE AND
HOW TO AUTOMATE THE PROCESSES.
THIS IS SHOWING THE SPACE THE
ASPIRE TEAM WAS IN BEFORE WE GOT
CLEARANCE TO START CONSTRUCTION.
ONE SILVER LINING OF THE
PANDEMIC SINCE WE HAD TO SEND
EVERYONE HOME WE HAD TO START
CONSTRUCTION.
ONE THING THAT WENT TO PLAN
DURING THE COVID-19 PERIOD WAS
OUR CONSTRUCTION.
THIS WAS COMPLETION AROUND 2021.
AGAIN WE WERE VERY EXCITED ABOUT
TO HAVE OUR LAB SPACE, NICE OPEN
LABS.
IF YOU WANT TO ADVANCE ONE MORE
SLIDE A YEAR LATER AND THE
CURRENT STATE.
PART OF THE REASON THE LAB IS SO
POPULATED ALL THING NCATS WE
HAVE TO BE DYNAMIC.
A LOT OF THE EQUIPMENT IS PART
OF THE ANTIVIRAL PREVENTION
PROGRAM AND THAT WAS A RESPONSE
FROM THE COVID PANDEMIC.
WE NEEDED LAB SPACE QUICKLY.
WE WERE ABLE TO PIVOT FAST AND
OCCUPY THAT AND IN THE BACK END
OF THE PITCH YOU SEE THE ASPIRE
AND PHYSICAL MODULES COMING TO
LIFE FOR THE PROGRAM.
WHAT WE'RE LOOKING TO DO IS
AUTONOMOUS ASSAY AND CHEMICAL
SYNTHESIS.
THIS IS AN AREA NCATS HAS
EXPERIENCE IN AND GREENS ARE
INPUTS AND BLUE IS OUTPUTS AND
THEY ACT AS INPUT FOR HIGH
THROUGHPUT AND VIRTUAL SCREENS
AND WE'VE HAD SUCCESS.
THE DATA IS ANALYZED TO
DETERMINE OPTIMAL ASSAY
CONDITIONS AND GENERATE RESPONSE
CURVES YOU SEE IN THE DATA
ANALYSIS PIECE.
THE RESULTING DATA IS USED TO
DETERMINE POTENTIAL MOLECULES TO
BE SYNTHESIZED AND IT'S AN
ITERATIVE CYCLE AND WE'RE
WORKING ON THE CHEMICAL
SYNTHESIS AND WOULD HAVE STEPS
LATER TO AUTOMATE CHEMICAL
SYNTHESIS THERE.
AND THE EXTRAMURAL PROJECT HELPS
BUILD A MORE INNOVATIVE AND
ROBUST PLATFORM THROUGH TEAM
SCIENCE TO ENABLE THE CHEMISTRY
DODA TALKED ABOUT AND TALKED
ABOUT THE FIVE AWARDEES.
THE FIVE AWARDEES AREN'T
EXISTING IN A VACUUM.
THEY'RE FILLING KEY ROLES AND
THE FIRST AWARDEES WAS PURDUE
UNIVERSITY WITH DR. GRAM COOKS
WITH THE GOAL OF TRANSFERRING
HARDWARE TO NCATS IN THE SECOND
PHASE FOR REACTION SCREENING AND
FOR AUTOMATED REACTIONS AND
BIOLOGICAL ASSAYS.
THAT FITS IN WELL WITH OUR ASSAY
OPTIMIZATION.
THE SECOND AWARDEE REFERENCED
THIS IS DR. CHOPRA AND LEDGER
FOR INTERACTIVE QUERY EXECUTION.
A BLOCK CHAIN FOR AUTONOMOUS
AUTOMATION AND FITS IN WELL WITH
EXISTING NCATS CAPABLEABILITY --
CAPABILITIES AND HAVE COMPLETE
TRACEABLE FOR EVERY STEP AND
FUNCTION.
WE HAVE INITIATED A PILOT WE'RE
INVOLVED IN A PROJECT PILOT TO
KICK IT OFF AND HAVE THE BLOCK
CHAIN CONTROL INSTRUMENT AT
NCATS WITH BIOLOGICAL SCREENING
WHERE WE'RE FURTHER AHEAD.
THE THIRD AWARDEE IS
COLLABORATIVE DRUG DISCOVERY AND
A VIRTUAL MODEL FOR APPROACHES
TO NEW CHEMISTRIES AND
COLLABORATIVE INFORMATICS.
USING MACHINE LEARNING TO
DEVELOP NOVEL CHEMISTRIES TO
PLUG IN WITH OUR CHEMISTRY ROUTE
PLANNING AND AUTOMATED CHEMICAL
SYNTHESIS.
WE HAVE M.I.T., DR. CONNOR
COOLEY FOR VIRTUAL MODULES TO
ENABLING NEW TECHNIQUES FOR
AUTONOMOUS OPERATION FOR
MULTISTEP CHEMICAL SYNTHESIS AND
MOLECULES.
WHAT'S EXCITING ABOUT THIS PIECE
IS IT CAME WITH TWO COMPONENTS
THAT HAVE GOTTEN SUBSTANTIAL
FUNDING THE OPEN REACTION
DATABASE WHICH IS AN INITIATIVE
SUPPORT LEARNING AND PREDICTION
CHEMICAL SYNTHESIS AND
EXPERIMENTAL DESIGN AND IN USE
BY THOUSAND OF RESEARCHERS AND
LINKED WITH PUB CHEM.
THAT'S ONE AREA THEY CAME WITH
FUNDING FOR THE SYNTHESIS
PLANNING MODULE OVER YEARS IN
INITIATIVE WITH DARPA.
WE'RE EXCITED BECAUSE THEY'RE
BRINGING THEIR OWN CAPABILITIES
AND ESSENTIALLY BUILDING
DISTRIBUTIVE NETWORK OF
INFORMATICS FOR THE RESEARCH
COMMUNITY AT LARGE.
AND THE UNIVERSITY WITH THREE
INDEPENDENT COMPONENTS.
DIGITIZE CHEMICAL STANDARD THEY
CALL KYDL AND YOU DON'T HAVE THE
VISUAL OF TAKE THE NOTEBOOK AND
THE CHEMICAL OR THE LABORATORY
NOTEBOOK AND THE SCROLL TO
ESSENTIALLY A FORMATTED COMPUTER
PROGRAMMING LANGUAGE TO TAKE
THAT LANGUAGE TO BE EXECUTED ON
THEIR COMPUTER WHICH IS A
UNIVERSAL CHEMICAL SYNTHESIS
DEVICE AND FINALLY HOW TO HAVE
THE OUTPUT OF THE MOLECULES
PRODUCED TO CREATE CHEMICAL
DISCOVERIES THROUGH MACHINE
LEARNING ALGORITHMS.
WE'RE EXCITED BECAUSE WE'RE
STARTING THE PROCESS OF BUILDING
A COMPUTER THAT WILL BE
INSTALLED IN THE NCATS
LABORATORY AND ONE EXPERIENCE WE
WANT TO RUN IS TO UTILIZE THE
BLOCK CHAIN BASED APPROACH FROM
THE LINK PROJECT TO INITIATE A
CHEMICAL SYNTHESIS TO RUN AT
NCATS AND GLASGOW AT THE SAME
TIME AND COMPARE RESULTS AND
FEED THEM BACK TO OUR
COMPUTATIONAL PLATFORM.
THAT'S AT A HIGH LEVEL.
WE'RE EXCITED ABOUT THAT WORK.
WHAT'S MORE EXCITING IS THE
TIMING OF THE PRESENTATION IS
GOOD BECAUSE MONDAY THE FIVE
GROUPS ARE SHOWING UP TO NCATS
IN PERSON AND THEY'VE ALL AGREED
AND SIGNED A FIVE-WAY RCA SO
THEY CAN ALL SPEAK FREELY ABOUT
WAYS TO COLLABORATE TOGETHER.
THOUGH THEY'RE SPECIFIC PIECES I
WANT TO MENTION THESE PIECES
AREN'T FIXED AND RIGID IN PLACE
TO ACROSS THE SPECTRUM BECAUSE
THIS IS SUCH A BROAD AREA OF
RESEARCH WE NEED ALL THE HELP WE
CAN GET.
THE CONTINUED GROWTH AND
CAPABILITY.
THE CURRENT CAPABILITY ARE
ADDITIONS TO AUTOMATION AND
MOLECULES USING AUTOMATED
PROCESSES, NOVEL ANALYTICAL
TECHNIQUES ON WHAT WAS MADE AND
DATA CAPTURE ACROSS PROCESSES
AND DATA SHARING AT SCALE WHICH
I THINK NCATS HAS LED IN THE
SPACE FOR SOME TIME AND
HIGHLIGHTED THAT THROUGH
INITIATIVES SUCH AS RDCRN AND
N3C.
THIS IS A NEXT STEP UP NOT
VIRTUAL BUT I THINK THIS GOES
BACK TO DODA NOW.
>>WE PROVIDED UPDATES ABOUT THE
ASPIRE PROGRAM.
I WANTED TO GIVE YOU SOME IDEA
WHERE WE THINK ABOUT FUTURE.
WE'D LIKE TO EXPAND OUR PROGRAM
TO INCLUDE COLLABORATIONS WITH
OTHER PROGRAMS SUCH AS 3-D BIO
PRINTING AND TISSUE CHIPS AND
WOULD LIKE TO EXPLORE CHEMICAL
SPACE WITH PRODUCT LIBRARIES AND
TO EXPLORE HOW WE CAN TARGET
OTHER BIOLOGICAL TARGETS SUCH AS
RNA.
AND ALSO SPACE TO INCLUDE
SYNTHETIC BIOLOGY AND EXPAND
CHEMICAL SPACE.
SO FAR WE HAD A FRUITFUL
COLLABORATION WITH DARPA AND
WOULD LIKE TO EXPAND WITH OTHER
I.C.s AND FEDERAL AGENCIES.
WHILE THE MAJORITY OF THE PEOPLE
WE'RE WORKING SO FAR WITH ARE
INTERESTED IN AUTOMATION WE'D
LIKE TO ENGAGE THOSE CHEMISTS
THAT AMENABLE TO AUTOMATION AND
USE ADDITIONAL FUNDING
MECHANISMS IN ORDER TO SPUR
PRIVATE SECTOR RELATIONS.
I'D LIKE TO TAKE TIME TO THANK
MANY PEOPLE WHO HAVE HELPED US
DEVELOP THESE PROGRAMS AND
EXECUTE THE PROGRAMS.
[LISTING NAMES]
SHOW HELPED DURING THE
INITIATIVES.
OUR MANAGER AND OUR GRANTS
MANAGEMENT TEAM AND THE BUDGET
TEAM AND THESE CONTRIBUTED TO
THE ASPIRE LABORATORY AND THE
TEAM FOR THEIR EXPERTISE AND
DEVELOPING FRUITFUL
COLLABORATIONS AND THE TEAM FOR
THE HEAL INITIATIVE AND OTHER
COLLEAGUES AND I.C.s FOR SPECIAL
MATTER EXPERTS AND FOR THE DARPA
TEAM.
THEY WERE OPEN IN SHARING THEIR
EXPERIENCES THEY HAVE OBTAINED
FROM THEIR PROGRAMS SO WE CAN
SUCCESSFULLY BUILD ASPIRE HERE.
THANK YOU VERY MUCH AND WE'RE
HAPPY TO ANSWER ANY QUESTIONS.
>>ANY QUESTIONS FOR DAN AND/OR
ASPIRE?
THAT WAS A PRETTY TECHNICAL
PRESENTATION.
IT MAY TAKE A BIT TO ABSORB BUT
IT'S A LARGE PROGRAM WE'RE
CONTINUING TO BUILD AND HAVE
BEEN BUILDING FOR THE LAST FIVE
YEARS AND WE'RE REALLY EXCITED
TO SEE THE ACTUAL LABS NOW BEING
BUILT OUT AND THE AWARDEES BEING
FUNDED IN PULLING THESE
TOGETHER.
I THINK THERE'LL BE MUCH MORE
TANGIBLE INFORMATION THAT WILL
BE COMING OUT IN SHORT ORDER.
>>IS THAT THE FACILITY IN
ROCKVILLE THE SLIDES WE IS A --
>>AT 9800 AND OUR NCATS
FACILITY.
SHADY GROVE.
>>YES.
ANY OTHER THOUGHTS OR QUESTIONS?
OKAY.
MATIAS.
>>VERY IMPRESSIVE.
AND THERE'S A SIGNIFICANT EFFORT
IN PLACE THAT CAN DEVELOP INTO
PRIVATE SECTOR AS WELL.
THIS IS A START UP IN BIO TECH
SPACE.
HAVE YOU ANY EXPLORATION WITH
LARGE PHARMA WITH STRATEGIC
INVESTMENT IN THE SPACE GOING
FORWARD TO SEE IF SOME ALIGNS IF
LARGE PHARMA COULD BE IN THE
CO-DEVELOPMENT OR CONVERSELY
GENERATING THE LARGE LIBRARIES
TO FIND A WAY TO HAVE THE
INVESTMENT COME BACK AND
UTILIZED WITH BIG PHARMA AS
WELL.
>>GREAT QUESTION.
WE HAVE COLLABORATIVE PROJECTS
WITH LARGE PHARMA OR A COMPANY I
MENTIONED FOR THE DEVELOPMENT OF
WHAT'S CALLED THE REACTOR AN
EIGHT-CHANNEL CHEMICAL SYNTHESIS
DEVICE.
THAT WAS ALSO IN CONCERT WITH
ELI LILLY.
WE VIEW SOME AREAS AS
PRE-COMPETITIVE DEVELOPMENT TO
AUTOMATE CHEMISTRY TAKE A STEP
BACK WITH THE FANCY STUFF IN THE
CLOUD AND AMIL.
IF WE CAN'T AUTOMATE CHEM
SYNTHESIS WE'RE NOT GOING
ANYWHERE.
IT'S THE SPACE.
WE WORKED WITH PARTNERS AND DO
LIKE THE OFFICE OF STRATEGY
ALLIANCE HELPED PUT THAT IN
PLACE STRATEGICALLY WHERE WE CAN
WORK TOGETHER WITH INDUSTRY
PARTNERS AND ALSO HAVE A CREATE
UP WITH OTHER.
AND WE USE MASS SPECT
TECHNOLOGY.
OPEN TO WORKING WITH PHARMA AND
THERE'S A LOT OF COLLABORATION
THOUGH WE'RE NOT DIRECTLY
WORKING WITH PHARMA THERE'S
EXCHANGE OF IDEAS AS A
RELATIVELY SMALL COMMUNITY.
WE'RE WORKING FORMAL
COLLABORATION AND IN NORMAL --
INFORMAL KNOWLEDGE SHARING TO
ENHANCE THE SPACE OF AUTO
CHEMISTRY.
>>THANK YOU, SAM.
>>I KNOW WITH NCATS YOU TAKE A
DISEASE AGNOSTIC APPROACH BUT IN
TERMS OF TRYING TO PILOT THIS,
IS THERE A PARTICULAR DISEASE
THAT YOU SEE INITIALLY SHOWING
THE FEASIBILITY WITH?
AND THEN MY OTHER QUESTION IS I
GUESS YOU MENTIONED FOR
SCREENING DO YOU SEE IT BEING
USED FURTHER DRUG EXTREME IN A
DRUG DISCOVERY PATHWAY OR HOW
FAR DOWN ?
>>WE OPEN IT UP TO ANY DRUG
TARGET AND WE HAVE INTEREST IN
RARE DISEASE AND THE TYPE OF
PROJECT YOU CAN ENVISION WILL BE
TREMENDOUS HELP TO DISCOVER NEW
TREATMENTS FOR RARE DISEASES.
EVENTUALLY IT WILL BE A PLATFORM
OPEN SOURCED AND ANYONE WITH A
TRANSLATIONAL PROBLEM WILL SAY I
HAVE THIS STAR GET WHAT
MOLECULES DO YOU SUGGEST TO
TARGET IT?
THIS IS DOWN THE ROAD WE
ENVISION IT AS A TREMENDOUS
RESOURCE FOR THOSE DISEASES THAT
INDUSTRY MAY NOT BE PRIMARILY
INTERESTED IN.
AND WITH REGARD TO THE SECOND
QUESTION, I THINK SAM MAY BE
BETTER IN ANSWERING THAT ONE.
>>AND SO I DO THINK THIS IS
GOING TO BE -- IT'S A GREAT
QUESTION BECAUSE FROM A
SCREENING PERSPECTIVE THAT
BEGETS IT WILL BE USED FOR PRE
CLINICAL DRUG DEVELOPMENT.
BUT IF WE THINK OF SYNTHESIS AND
SCALE UP OF ACTUAL PRODUCT, WILL
THAT HAVE FURTHER USE DOWN
STREAM?
WE HAVEN'T REALLY TALKED ABOUT
THE LEVEL OF COULD THIS BE USED
TO CREATE GMP GRADE
PHARMACEUTICALS.
IT'S NOT REALLY OUR FOCUS AT
NCATS TO SAY IT COULDN'T IF
THERE THERE'S A NEED BUT IT DOES
RESIDE IN THE PRE-CLINICAL AREA
ESPECIALLY BECAUSE IT'S HOUSED
WITHIN THE INTRAMURAL PROGRAM
AND THAT'S THE FOCUS OF DPI AND
WHEN THIS FIRST GOT KICKED OFF
WITH THIS COULDN'T HAVE HAPPENED
WITHOUT OUR SCIENTIFIC DIRECTOR.
THE INTENDED PURPOSE IS
PRECLINICAL BUT THE TECHNOLOGIES
AND TECHNIQUES INVOLVED COULD BE
APPLIED TOWARDS DOWNSTREAM MORE
CLINICAL APPROACHES.
>>THANKS.
AND TO THE FIRST PART OF YOUR
QUESTION, WHAT HELPED KICK OFF
THE PROGRAM IS WORK WITH THE
HEAL PROGRAM HELPING END
ADDICTION LONG-TERM.
WE STARTED WITH THAT FOCUS AND
AS DOBA MENTIONED WE'RE
INTERESTED IN WHAT WE DO NEXT A
LITTLE BIT MORE THAT'S BEEN AN
AREA OF INTEREST BECAUSE WE'VE
WORKED CLOSELY WITH THE GROUP
AND IT'S AN UNMET NEED AS WELL.
>>I FORGOT THAT'S WHERE IT
STARTED.
THAT ANSWERS THE QUESTION.
THANKS.
>>OKAY.
ANY OTHER QUESTIONS?
>>ANNA, I THIS CONCLUDES THE
PRESENTATIONS FOR THE DAY.
IT'S BEEN A SHORT MEETING BUT A
LONG DAY AND A LOT OF
PRESENTATIONS, A LOT OF DATA
THROWN AT YOU BUT I HOPE YOU
SENSE THE EXCITEMENT OF WHERE
WE'RE GOING AND WILL STAY TUNED
TO UNDERSTAND HOW WE'RE GOING TO
BE PUSHING FORWARD ON THE
STRATEGIC PLANNING PROCESSES AND
WE'LL BE REACHING OUT IT A
VARIETY OF FOLKS AND YOU'LL BE
AMONGST THEM SO STAY TUNED ON
THAT.
AND WITH THAT UNLESS THERE'S
ANYTHING ELSE YOU WANT TO BRING
UP I'LL GIVE IT ONE LAST
OPENING -- TIME -- OKAY.
I'M GOING GAVEL US OUT THEN AND
DO THIS AWKWARD THING ABOUT
MOVING THE VIDEO.
THANK YOU SO MUCH.
THIS CONCLUDES OUR MEETING.
>>THANKS, EVERYONE.