GOOD MORNING, I'M JENNIFER
MORTON GRAY THE CHIEF OF THE
COMMUNICATIONS AND OUTREACH
BRANCH AT NIAMS AND IT'S MY
ABSOLUTE PLEASURE TO SEE YOU ALL
HERE TODAY AND WELCOME TO YOU
THE 2023 NIAMS COALITION
OUTREACH AND EDUCATION MEETING.
THAT DESERVES A ROUND OF
APPLAUSE.
WE'RE DELIGHTED THAT ABOUT 55 OF
OUR COLLEAGUES REPRESENTING MORE
THAN 45 ORGANIZATIONS ARE HERE
TODAY.
AS YOU KNOW, THE NIAMS PORTFOLIO
IS BROAD INCLUDING DISEASES THAT
AFFECT THE BONES, JOINTS,
MUSCLES AND SKIN.
THESE DISEASES AFFECT ALMOST
EVERY HOUSEHOLD IN AMERICA,
PEOPLE OF ALL AGES, ALL RACIAL
AND ETHNIC POPULATIONS AND EVERY
ECONOMIC STRATA.
AS WE WORK TO LEARN MORE ABOUT
THESE DISEASES, THE NIAMS
COALITION AND IT'S NEARLY 100
MEMBER ORGANIZATION HAVE BEEN
TREMENDOUS ALLIES OF NIAMS AND
THE NIH.
WE VALUE BRINGING THIS GROUP
TOGETHER EVERY TWO YEARS FOR
THIS MEETING AND LOOK FORWARD TO
CONTINUING TO WORK WITH YOU TO
BUILD ON OUR COMMON GOAL OF
TURNING DISCOVERY INTO PUBLIC
HEALTH.
TODAY WE HAVE A GREAT PROGRAM IN
STORE.
YOU'LL HEAR FROM INFLUENTIAL
LEADERS FROM ACROSS NIH PROGRAMS
AND BEYOND AS WELL AS
INFORMATIVE BREAKOUT SESSIONS
LED BY NIAMS' PROGRAM DIRECTORS
AND YOUR OWN COALITION
COLLEAGUES.
THIS MEETING IS INTERACTIVE.
DUE TO THE AMOUNT OF NETWORKING
ACTIVITIES AND SESSION WE BUILT
IN THE AGENDA WE'LL BE MOVING TO
VARIOUS ROOMS AND ORGANIZING
INTO GROUPS THROUGHOUT THE DAY.
THERE WILL ALSO BE OPPORTUNITIES
TO NETWORK AND LEARN FROM ONE
ANOTHER AT THE POSTER SESSION
AND DURING BREAKS AND LUNCH.
SO BEFORE WE BEGIN, I WOULD
FIRST LIKE TO ACKNOWLEDGE THE
NIAMS LEADERSHIP AND STAFF WHO
ARE HERE TODAY.
IT'S WONDERFUL TO SEE ALL OF
YOU.
WE WOULD ALSO LIKE TO
ACKNOWLEDGE THE TIRELESS WORK OF
THE COALITION PLANNING
COMMITTEE, TRISHA, EVAN AND
LAUREN WHO HELPED PLAN FOR THIS
MEETING.
I'D ALSO LIKE TO RECOGNIZE THE
NIAMS STAFF WHO WORKS WITH THE
LEADERSHIP TO PUT TOGETHER THE
PROGRAM.
IT'S WAS A COLLABORATIVE TEAM
EFFORT BUT SPECIAL THANKS GO TO
COLLEEN, SARA, LESLIE, BEATA AND
COLLEEN WHO LED THE CHARGE.
I WANT TO EXTEND OUR
APPRECIATION OUR CONTRACT
SUPPORT COLLEAGUES LED MY NATE
ROBINSON INSTRUMENTAL IN THE
SUCCESS AND MANY THANKS TO THE
CORRELATION ORGANIZATIONS WHO
PROVIDED BREAKFAST, LUNCH AND
REFRESHMANMENTSS -- REFRESHMENS
THROUGHOUT THE DAY AND NOW
COLLEEN LENDA.
>> GOOD MORNING, EVERYONE.
IT'S SO WONDERFUL TO SEE SO MANY
FAMILIAR FACES AND SO OF YOU WE
HAVEN'T MET IN PERSON AND IT'S
JUST SO NICE TO SEE EVERYONE IN
PERSON.
I'M GOING TO COVER A FEW
HOUSEKEEPING ITEMS AND
TRANSITION TO OUR PLENARY
SESSION.
A REMINDER THIS IS RECORD.
IF YOU DIDN'T WANT YOUR PHOTO
TAKEN OR ANY RECORDING YOU
SHOULD HAVE RECEIVED SOMETHING
ON YOUR BADGE WHEN YOU CAME IN
SO STOP AT REGISTRATION IF NOT.
ADDITIONALLY, IF YOU COULD JUST
MAKE SURE YOU'RE PHONE IS ON
SILENT FOR OUR SESSIONS, THAT
WOULD BE TERRIFIC.
THE BATHROOMS ARE ON THE RIGHT
AND LEFT-HAND SIDE OF THE BACK
OF THE MEETING SPACE.
NEXT YOU'LL NOTICE TENT CARDS ON
EACH OF THE TABLE.
USE THE QR CODES TO CONNECT AND
ENGAGE.
WE'RE USING THE HASH TAG NIAMS
COALITION DAY AND GO ON SOCIAL
MEDIA AND SHARE ABOUT THE
MEETING, TAKE PHOTOS WITH EACH
OTHER AND SHARE AS MUCH AS YOU
CAN AND WE'LL HAVE IN YOUR
PACKET AN EVALUATION FORM WHICH
IS GOING TO HELP US TO LEARN
MORE ABOUT HOW YOU FEEL ABOUT
EACH OF THE SESSIONS AND WE DO
PAY ATTENTION TO THAT FEEDBACK
SO PLEASE TAKE SOME TIME TO FILL
THAT OUT AND YOU CAN RETURN IT
TO THE REGISTRATION DESK AT THE
ENDS OF THE DAY.
-- END OF THE DAY.
FOR Q&A THROUGHOUT THE DAY IN
THIS ROOM IF YOU HAVE A QUESTION
JUST RAISE YOUR HAND AND WE HAVE
WONDERFUL STAFF ON THE SIDELINE
ARE GOING TO BRING THE
MICROPHONE OVER FOR YOU.
DON'T NEED TO MOVE AT ALL.
FOR THE BREAKOUT SESSIONS WHICH
WILL BE IN SMALLER ROOMS YOU'RE
NOT GOING IT NEED ANY
MICROPHONES SO YOU CAN ASK YOUR
QUESTION IN SPACE.
THEY'RE SMALLER SPACES.
NOW THE HOUSEKEEPING IS TAKEN
CARE OF I'M PLEASED TO INTRODUCE
TRISHA AND LEE GROSSMAN.
TRISHA WORKS WITH VARIOUS
STAKEHOLDERS TO ADVANCE THE
MISSION TO SUPPORT PATIENTS AND
ACCELERATE RESEARCH.
SHE CURRENTLY SERVES ON DREXEL
UNIVERSITY'S HEALTH
ADMINISTRATION DEPARTMENT
ADVISORY COUNCIL AND STARING
COMMITTEE.
SHE DEDICATED HER CAREER TO
IMPROVING THE LIVES WITH THOSE
WITH RARE DISEASE AND WITH OTHER
CORRELATION WORK HERE.
THE CO-CHAIR LEE GROSSMAN BUILDS
IMPORTANT PARTNERSHIP TO ADVANCE
THE OREFs MISSION.
SHE'S -- HE'S A PRESENTER ON
CHRONIC HEALTH CONDITIONS AND
MENTAL HEALTH.
IT'S BEEN AN ABSOLUTE HONOR
WORKING WITH TRISHA AND LEE AND
OTHER COALITION MEMBERS OVER THE
PAST SIX YEARS.
I'M NOW GOING TO TURN IT OVER TO
TRISHA AND LEE TO INTRODUCE OUR
FIRST SPEAKER.
>> SO WELCOME EVERYONE.
WE'RE SO EXCITED TO SEE
EVERYBODY HERE.
WE'RE HOPE YOU'LL HAVE
CONNECTIONS BY THE END OF THE
DAY.
HOPEFULLY YOU'LL KNOW LOTS OF
PEOPLE YOU DIDN'T BEFORE AND BE
ABLE TO BUILD THAT OUT.
WE'RE NOT GOING TO TAKE UP TOO
MUCH TIME BECAUSE WE'LL BE
PRESENTING LATER BUT WELCOME TO
YOU ALL.
WE'RE EXCITED TO GET STARTED AND
WITH THAT I'M GOING TO PASS IT
OVER TO LEE WHO WILL INTRODUCE
OUR FIRST SPEAKER.
>> THROUGHOUT THIS MORNING
YOU'LL SEE THAT TRISHA ANDRY
DOING TAG TEAM QUITE A BIT SO
WE'LL BE GOING BACK AND FORTH
BUT GOOD MORNING EVERYONE.
IT'S WONDERFUL TO SEE YOU HERE.
IT'S BEEN A LONG HAUL OVER THE
LAST FEW YEARS.
LIFE HAS CHANGED DRAMATICALLY
AND IT'S GREAT TO HAVE US ALL
HERE IN PERSON AGAIN.
HAVE THE UNIQUE HONOR OF
INTRODUCING OUR FIRST SPEAKER
TODAY AND THAT'S DR. LINDSEY
CRISWELL THE DIRECTOR OF NIAMS
AND WILL PROVIDE THE STATE OF
THE INSTITUTE.
SHE'S OVERSEES THE INSTITUTE'S
ANNUAL BUDGET OF MORE THAN $685
MILLION WHICH SUPPORTS RESEARCH
INTO THE CAUSES, PREVENTION OF
ARTHRITIS AND MUSCULOSKELETAL
SKIN DISEASES.
SHE WAS AT CALIFORNIA SAN
FRANCISCO AND IS A CERTIFIED
RHEUMATOLOGIST AND SHE IS A
PRINCIPLE INVESTIGATOR OF
MULTIPLE NIH GRANTS AND
PUBLISHED MORE THAN 250
PEER-REVIEWED PAPERS.
HER RESEARCH FOCUSES ON THE
GENETICS AND EPIDEMIOLOGY OF
HUMAN AUTOIMMUNE DISEASE AND IN
2021 WAS ELECTED TO THE
ASSOCIATION OF AMERICAN
PHYSICIANS, AN HONOR EXTENDED TO
PHYSICIANS WITH OUTSTANDING
CREDENTIALS IN BIOMEDICAL
RESEARCH.
ON A PERSONAL NOTE I'VE BEEN TO
NUMEROUS ORTHOPEDIC MEETINGS
THIS YEAR AND HAVE BEEN ABLE TO
ENGAGE WITH DR. CRISWELL AND
FIND HER EXTREMELY ENGAGING AND
OPEN AND ACCESSIBLE.
THESE ARE TREMENDOUS TRAITS FOR
ANY LEADER AND ONE OF THE
REASONS WHY SHE MAKES SUCH A
WONDERFUL PARTNER TO THE NIAMS
COALITION.
SO ON BEHALF OF THE NIAMS
COALITION, PLEASE ACCEPT OUR
WARM WELCOME AND WE ARE ANXIOUS
TO HEAR THE STATE OF THE
INSTITUTE.
THANK YOU.
>> SO, WELL, MORNING.
AND LET ME SEE -- I DON'T WANT
DO CREATE BIG ECHO.
VERY NICE TO SEE YOU ALL.
MY FIRST IN PERSON OUTREACH AND
EDUCATION DAY.
IT'S A PLEASURE TO MEET SOME OF
YOU I HAVEN'T HAD THE
OPPORTUNITY TO MEET IN PERSON
AND WE'RE GRATEFUL FOR YOUR
SUPPORT.
THANKS TO TRISHA AND LEE FOR THE
KIND INTRODUCTION.
IT'S AN HONOR TO BE GIVING THIS
STATE OF THE INSTITUTE ADDRESS
TODAY AND I'M DELIGHTED TO HAVE
ALL OF YOU HERE ON THE NIH
CAMPUS.
NEEDLESS TO SAY THIS MEETING IS
POSSIBLE DUE TO THE DEDICATED
EFFORTS OF NUMEROUS PEOPLE AND I
HAVE TIME TO MENTION ONLY A FEW
OF THEM HERE BUT WANTED TO
PARTICULARLY THANK MS. DUNNDAS
AND THE NIAMS COALITION
CO-CHAIRS YOU JUST HEARD FROM
TRISHA LEE AND LEE GROSSMAN.
I'D LIKE TO THANK ALL MEMBERS OF
THE ORGANIZING COMMITTEE AND
CO-SPONSORS FOR THEIR
GENEROSITY.
I APPRECIATE THE STEERING
COMMITTEE MEMBERS.
I KNOW YOU PLAY A CRITICAL ROLE
IN SUPPORTING THE CO-CHAIRS AND
LEADING THE COALITION.
I'LL DO A QUICK CHECK THE SOUND
IS OKAY.
IT SOUNDS ECHOEY UP HERE BUT
OKAY IN THE BACK?
ALL RIGHT.
SO MY TALK TODAY IS ORGANIZED
INTO TWO PARTS.
I'LL START WITH A BRIEF
INTRODUCTION TO THE NIH AS I
UNDERSTAND SOME OF THE GROUPS
ARE NEW TO THE NIAMS COALITION
FOLLOWED BY AN UPDATE ON THE NIH
AND NIAMS BUDGETS BUT THE
MAJORITY WILL PROVIDE AN
OVERVIEW OF NIH AND NIAMS
ACTIVES OF POTENTIAL INTEREST TO
YOU.
AS YOU MIGHT KNOW, NIAMS IS ONE
OF 27 INSTITUTES AND CENTERS
THAT COMPRISE THE NIH.
AND I CONSIDER THESE SIBLING
INSTITUTES AND CENTERS AS AN
INVALUABLE ASSET AND WE'RE
LOOKING TO ESTABLISH PARTNERSHIP
AND LEVERAGE OPPORTUNITIES OF
COMMON INTEREST.
LIKE THE REST NIH, NIAMS INVESTS
MOST OF THE MONEY AT
INSTITUTIONS AROUND THE COUNTRY
APPROXIMATELY 11% GOES IT OUR
INTRAMURAL RESEARCH PROGRAM HERE
IN BETHESDA AND THE SLICE YOU
SEE RMS, RESEARCH AND MANAGEMENT
SUPPORT IS WHAT YOUR
ORGANIZATIONS WOULD CALL
OVERHEAD.
YOU CAN SEE WITH THE TWO PIE
CHARTS THE INSTITUTE'S
INVESTMENT IN INTRAMURAL
RESEARCH AND THE RMS EXPENSES
ARE ON PAR WITH THE ENTIRE NIH
AND THE EXTRAMURAL INVESTMENTS
SLIGHTLY LARGER.
SO THIS SLIDE BRIEFLY EXPLAINS
HOW THE NIH EXTRAMURAL WITH
GRANT PROPOSALS ARE EVALUATED
WORKS.
THE APPLICANT DEVELOPS AN
ORIGINAL IDEA AND SUBMITS AND
THE EXPERTS REVIEW THE
APPLICATIONS.
NOW, THE FIRST LEVEL OF PEER
REVIEW IS ESSENTIAL FOR LETTING
INVESTIGATORS AND THE INSTITUTE
INFORMATION THE STRENGTHS AND
THE WEAKNESSES OF THE PROPOSED
PROJECTS.
BUT BEFORE AN INSTITUTE OR
CENTER CAN FUND AN APPLICATION,
IT MUST UNDER GO A SECOND LEVEL
OF PEER REVIEW BY ITS ADVISORY
COUNCIL.
ADVISORY COUNCIL MEMBERS ARE
APPOINTED BY THE SECRETARY OF
THE DEPARTMENT OF HUMAN HEALTH
AND SERVICES AND INCLUDE MEMBERS
OF THE PUBLIC SUCH AS
REPRESENTATIVES OF
PATIENT-FOCUSSED ORGANIZATIONS.
AND SCIENTIFIC AND LAY MEMBERS
ARE SELECTED FOR DIVERSITY OF
INTEREST, WIDE GEOGRAPHICAL
REPRESENTATION AND THE ABILITY
TO TAKE IN THE ISSUES THAT COME
BEFORE THE BODY.
SO NOW MOVING ON TO THE NIH AND
NIAMS BUDGET.
ON MARCH 9 PRESIDENT BIDEN
RELEASED HIS FISCAL YEAR 2024
BUDGET REQUEST TO CONGRESS.
THE AMOUNT FOR NIH IS $51.1
BILLION AN INCREASE OF 3.9% OVER
THE FISCAL YEAR 2023 ENACTED
BUDGET.
FOR NIAMS THE AMOUNT IS $688
MILLION WHICH REPRESENTS NO
CHANGE FROM FISCAL YEAR 2023.
THAT'S THE CURRENT YEAR.
NOW, SINCE THEN THE HOUSE AND
SENATE HAVE RELEASED THEIR OWN
BUDGET PROPOSALS.
AND IN A TIME WHEN MANY AGENCIES
ARE FACING THE PROSPECT OF
BUDGET CUTS WE'RE GRATEFUL TO
THE AMERICAN PUBLIC AND OUR
ELECTED OFFICIALS FOR KEEPING
ARTHRITIS AND MUSCULOSKELETAL
AND SKIN DISEASES AS A PRIORITY.
AS NEXT STEP THE HOUSE AND
SENATE MUST CONVENE A CONFERENCE
COMMITTEE WHERE THEY AGREE ON
NUMBERS NOT JUST FOR NIH BUT THE
ENTIRE DEPARTMENT OF HEALTH AND
HUMAN SERVICES AS WELL AS THE
DEPARTMENTS OF LABOR AND
EDUCATION.
AND THEN THAT VERSION OF THE
BILL MUST PASS BOTH CHAMBERS
BEFORE GOING TO THE PRESIDENT
FOR SIGNATURE.
NOW, THIS MAY OR MAY NOT OCCUR
BEFORE OUR CURRENT FISCAL YEAR
ENDS SEPTEMBER 30 SO WE WON'T BE
SURPRISED IF WE BEGIN FISCAL
2024 ON A CONTINUED RESOLUTION,
HOPEFULLY, AND THIS MEANS WE CAN
CONTINUE TO OPERATE AT OR
SLIGHTLY BELOW OUR FISCAL YEAR
2023 LEVEL.
SO WE'RE ALWAYS EXPLORING
OPPORTUNITIES TO BROADEN OUR
RESOURCES SUPPORTING ARTHRITIS
AND MUSCULOSKELETAL AND SKIN
DISEASE NOT IN BUT ALSO OUTSIDE
NIH.
IT'S A FABULOUS EXAMPLE OF ONE
PUBLIC-PRIVATE AMONG THE NIH,
FDA AND INDUSTRY AND NON-PROFIT
GROUPS INCLUDING SOME NIAMS
COALITION MEMBERS AND OTHER
ORGANIZATIONS.
OVER THE PAST EIGHT YEARS THE
AMP PROGRAM AND RM LUPUS HAS
MADE PROGRESS IN UNDERSTANDING
THE CELL POPULATIONS AN PATHWAYS
THAT DRIVE THE DISEASES AND WE
GOT INSIGHTS INTO TARGET
IDENTIFICATION AT EARLY DISEASE
STAGES, ENABLING ADDITIONAL
STUDIES USING THE BIO SPECIMENS
COLLECTED AND ADVANCING ANALYTIC
APPROACHES AND TECHNOLOGY.
SO BUILDING ON THIS SUCCESS, THE
AMP AUTOIMMUNE AND IMMUNE
MEDIATED PROGRAM WAS LAUNCHED IN
2021.
AMP AIM AS WE REFER TO IT
EXTENDS BEYOND RA AND LUPUS TO
SJOGREN'S DISEASE AND OTHER
DISEASES AND THERAPEUTIC TARGETS
AND BENCHMARK FOR THE MOLECULAR
CLASSIFICATION OF DISEASES, AMP
AIM WILL PUT THE PIECES BACK
TOGETHER TO DISCOVER HOW IMMUNE
CELLS AND CELLS IN THE TISSUES
INACT TO CAUSE INFLAMMATION AND
DISEASE AND ACCELERATE DISEASE
MECHANISMS AND THERAPEUTIC
TARGETS.
NOW, AS WITH AMP, RA AND LUPUS,
COLLABORATION AND TEAM SCIENCE
ARE KEY COMPONENTS OF AMP AIM.
THE PROGRAM BRINGS TOGETHER
MULTI-DISCIPLINARY RESEARCHERS
AND ENGAGES EARLY CAREER
INVESTIGATORS TO DEFINE SHARED
IMMUNE MECHANISMS AMONG THESE
DISORDERS AS WELL AS MECHANISMS
THAT MAY BE UNIQUE TO EACH
DISEASE.
SO WE EXPECT THIS WILL ENABLE
THE DEVELOPMENT OF NEW AND
EFFECTIVE THERAPIES FOR PEOPLE
WITH AUTO IMMUNE DISEASE AND
ADDRESSING CHRONIC DISEASE MORE
GENERALLY.
IMPORTANTLY, THE PROGRAM ALSO
HAS DEVELOPED THE ARTHRITIS AND
AUTOIMMUNE PORTAL TO ACCELERATE
SHARING IN THE RESEARCH
COMMUNITY.
FURTHER, WE THOUGHT YOU MIGHT BE
INTERESTED IN HEARING AN UPDATE
ABOUT THE BESPOKE GENE THERAPY
CONSORTIUM WHICH IS THE LATEST
AMP COMPONENT THAT WAS LAUNCHED
SHORTLY AFTER THE LAST MEETING
IN 2021.
SO BECAUSE GENE THERAPY HAS SUCH
POTENTIAL ACROSS DISEASE SPACES
AND DISORDERS, BDTC APPLICANTS
PROPOSE THE DISEASES TO BE
STUDIED.
SUCCESSFUL APPLICATIONS FOCUS ON
DISEASES AMONG OTHER
CHARACTERISTICS HAVE A CLEARLY
DEFINED PATIENT GROUP ALREADY
ASSEMBLED TO HELP MOVE POTENTIAL
THERAPIES INTO CLINICAL TESTING
AND THE GOAL IS TO GENERATE A
STREAMLINED CLINICAL AND
REGULATORY FRAMEWORK FOR GENE
THERAPY LEADING TO STANDARDIZED,
FASTER AND LESS EXPENSIVE
TREATMENT DEVELOPMENT INSTEAD OF
THE ONE-OFF APPROACHES USED FOR
CURRENT THERAPIES.
IN MAY, THE FOUNDATION FOR THE
NIH OR THE FNIH WHO MANAGES THE
AMP PROGRAM ANNOUNCED THE AMP
BGTC SELECTED EIGHT RARE
DISEASES FOR THE CLINICAL TRIAL
PORTFOLIO BY A PANEL OF EXPERTS
FOLLOWING RIGOROUS SCIENTIFIC
AND TECHNICAL REVIEW USING THE
BGTC ESTABLISHED CRITERIA
INCLUDING THE ADEQUACY OF THE
GENE FOR INSERTION INTO AN ADENO
ASSOCIATED VIRUS VECTOR,
SUFFICIENT PROOF OF CONCEPT AND
NATURAL HISTORY DATA, THE
EXISTENCE OF AN ESTABLISHED
DISEASE MODEL, LACK OF AVAILABLE
TREATMENT AND READINESS FOR
ENTERING INTO CLINICAL TRIAL.
AND AS SHOWN IN THE SLIDE AT
LEAST TWO OF THE DISEASES SELECT
ED ARE OF GREAT INTEREST TO THE
NIAMS COMMUNITY.
MORQUIRO SYNDROME IS A LYSOSOMAL
DISEASE OF CONDROITON AND
PROPIONIC HAS AN ENZYME INVOLVED
IN THE BREAKDOWN OF AMINNOW --
AMINO ACIDS WITH DIMINISHED
MUSCLE DONE AND DEHYDRATION AND
SEIZURES.
WITH THE SELECT OF THE
CONDITIONS BGTC WILL STREAMLINE
THE PROCESS TO DEVELOP THERAPIES
MORE GENERALIZABLE AND
ACCESSIBLE SO OTHERS CAN
BENEFIT.
WE'RE EXCITE ABOUT THE PROGRESS
AND LOOK FORWARD IT HEARING MORE
FROM DR. GERBERD IG THIS
MORNING.
AND THE AMP CELL BASED THERAPY
CONSORTIUM OR CBTC WILL LEVERAGE
THE BESPOKE GENE THERAPY MODEL.
RESPONSES TO THIS REQUEST
INFORMATION SHOULD BE SUBMITTED
BY SEPTEMBER 25 AND INFORMATION
GATHERED WILL BE DISCUSSED
FURTHER IN A TWO-HOUR WEBINAR
SEPTEMBER 28.
I WANTED TO MAKE SURE THOSE THAT
MAY BE INTERESTED ARE AWARE.
AS NOTED ON THE PROPOSED FISCAL
YEAR 2024 BUDGET SIDE THE
BUDGETS INCLUDE FUNDING FOR
ARPA-H.
ARPA STANDING FOR ADVANCED
RESEARCH PROJECT AGENCY AND THE
H FOR HEALTH IS BASED ON DARPA
WHICH IS THE DEFENSE ADVANCE
RESEARCH PROJECT AGENCY THE
CENTRAL RESEARCH AND DEVELOPMENT
AGENCY FOR THE DEPARTMENT OF
DEFENSE.
SO ARPA-H WOULD USE DARPA AS A
MODEL TO ESTABLISH A CULTURE OF
CHAMPIONING INNOVATIVE IDEAS IN
HEALTH AND MEDICINE BY FOCUSSING
ON MULTIPLE TIME-LIMITED
PROJECTS WITH DIFFERENT
APPROACHES TO ACHIEVE A
QUANTIFIABLE GOAL RELYING ON
DEFINED METRICS AND INJECTING
ACCOUNTABILITY THROUGH MEETING
THE METRICS AND SUPPORTING
USE-DRIVEN IDEAS FOCUSSED ON
SOLVING PRACTICAL PROBLEMS THAT
ADVANCE EQUITY AND RAPIDLY
TRANSFORM BREAKTHROUGHS INTO
TANGIBLE SOLUTIONS FOR ALL
PATIENTS.
ARPA-H ANNOUNCE THE THE FIRST
PROGRAM IN MAY THE NOVEL
REGENERATION FOR TISSUE
REGENERATION WHICH AIMS TO SOLVE
TREATMENT CHALLENGES IN OA.
WE WERE DELIGHTED TO SEE THIS
IDENTIFIED AS A PRIORITY.
NITRO WILL LOOK AT THREE AREAS.
IT WILL FUND SEVERAL RESEARCH
TEAMS TO DEVELOP AND LEVERAGE
INNOVATIVE FORMS OF REGENERATIVE
MEDICINE TO CREATE MINIMALLY
INVASIVE THERAPEUTICS THAT FULLY
REGENERATE DAMAGED JOINTS.
SINCE THE LAUNCH OF NITRO
THERE'S BEEN SEVERAL MORE
PROGRAMS ANNOUNCED FROM ARPA-H
AND HOPE YOU'LL HEAR THEM FROM
THE ARPA-H DIRECTOR WHO WILL BE
SPEAK TO THIS GROUP.
IN ADDITION TO THIS THERE'S
VOLUNTARY PATIENT ORGANIZATIONS
THROUGH THE MDCC THE
MUSCULODYSTROPHY CORE CENTER.
COMMITTEE MEETINGS ARE HELD
TWICE A YEAR AND OPEN TO THE
PUBLIC AND THE ARE WEBCAST FOR
THOSE WHO CAN'T ATTEND IN
PERSON.
TWO-THIRDS OF THE MEMBERS
REPRESENT GOVERNMENT AGENCIES
ONE-THIRD REPRESENT THE PUBLIC
AND PATIENTS, FAMILIES AND
PATIENT ADVOCATES ARE ALWAYS
WELCOME TO ATTEND MEETINGS AND
PROVIDE COMMENTS.
THE MEMBERSHIP HAS A BROAD RANGE
OF EXPERTISE ON MUSCULAR
DYSTROPHY AND THE ISSUES FACING
THE PROVIDERS AND PATIENTS AND
FAMILIES AND CURRENT VIEWS AND
STRATEGIES FOR THE GROUP.
LOOKING TO THE FUTURE, IT'S NOW
TIME TO RENEW THE ACTION PLAN
AND THE COMMITTEE HAS BEGUN
DISCUSSING A PROPOSED APPROACH
WHICH WAS PRESENTED AT THE MOST
RECENT COMMITTEE MEETING HELD IN
APRIL.
THE 2025 ACTION PLAN RENEWAL
WILL INCORPORATE A VARIETY OF
STAKEHOLDER PERSPECTIVES IN
ADDITION, THE COMMITTEE PLANS TO
INTEGRATE WORKFORCE TRAINING AND
CAREER DEVELOPMENT FOR
INDIVIDUALS AND LASTLY THE
COMMITTEE WOULD LIKE TO REFLECT
THE STRATEGY PLAN'S DEVELOPMENT
BY MDCC MEMBER ORGANIZATIONS AND
OTHER ADVOCACY GROUPS.
NOW WITHIN THE NIH, NIAMS
PARTICIPATES IN MANY
COLLABORATIVE EFFORTS WITH OTHER
INSTITUTES, CENTERS AND OFFICES
THAT HAVE SHARED INTERESTS.
THIS FISCAL YEAR, CONGRESSIONAL
KA
-- CONGRESS ALLOCATED $10
MILLION TO ESTABLISH THE OFFICE
OF AUTOIMMUNE DISEASE RESEARCH.
THE FUNDING WAS INCLUDED TO
IMPLEMENT THE RECOMMENDATIONS OF
A RECENT NASEM REPORT.
AS A LEADING FUNDER OF
AUTOIMMUNE DISEASES RESEARCH
NIAMS IS WORKING WITH THE NEW
OFFICE AS IT BEGINS TO
ACCOMPLISH ITS GOAL THE
MULTI-INSTITUTE AND RESEARCH
STRATEGIC PLAN AND IDENTIFYING
EMERGING AREAS OF INNOVATION AND
RESEARCH OPPORTUNITIES FOR THE
STRATEGY PLAN, COORDINATING AND
FOSTERING COLLABORATIVE RESEARCH
ACROSS THE NIH, ANNUALLY
EVALUATING THE NIH RESEARCH
PORTFOLIO AND PROVIDING
RESOURCES TO SUPPORT RESEARCH,
COLLABORATION AND DEVELOPING AND
OVERSEEING A PUBLICLY ACCESSIBLE
CENTRAL REPOSITORY FOR
AUTOIMMUNE RESEARCH RESULT.
IN LINE WITH THE NEW OFFICE, THE
OFFICE OF RESEARCH IN WOMEN'S
HEALTH AND OTHER INSTITUTES AND
CENTERS AND NIAMS PUBLISHED ON
THE EXPOSOME IN AUTOIMMUNE
DISEASE IT'S A COLLABORATING
TEAM PLANNING AWARDS EXACT PLAN
AND IT'S AN EFFORT BETWEEN THE
OFFICE OF RESEARCH ON WOMEN'S
HEALTH, THE NATIONAL INSTITUTE
OF ENVIRONMENTAL HEALTH SCIENCE
AND OTHER INSTITUTES AND CENTERS
ENGAGED IN AUTOIMMUNE DISEASE
RESEARCH.
THE GOAL IS TO INVITE
APPLICATIONS FOR EXPLORATORY,
EARLY AROUND CONCEPTUAL PLANNING
PROJECTS AIMED AT DEVELOPING A
FUTURE TEAM SCIENCE RESEARCH
NETWORK.
SO THE IDEA WOULD BE TO CONDUCT
RESEARCH TO DISCOVER THE
ENVIRONMENTAL EXPOSURES THAT
INFLUENCE DISEASE SUSCEPTIBLE,
ONSET AND OUTCOMES AND TO
DEVELOP NEW APPROACHES TO
UNDERSTANDING THE MECHANISMS
UNDERLYING HOW EXPOSURES PERTURB
CELLULAR, ORGAN AND TISSUE
FUNCTION ACROSS AUTOIMMUNE
DISEASES.
ANOTHER TRANS-NIH EFFORT I'D
LAKE TO HIGHLIGHT IS THE
MOLECULAR TRANSDUCERS OF
PHYSICAL ACTIVITY CONSORTIUM AS
THE EARLY RESULTS FROM THE
PROGRAM ARE FORTHCOMING.
MOTRPAC IS A COMMON FUND PROGRAM
LED BY NIAMS, THE NATIONAL
INSTITUTE OF AGING AND THE
NIDDK.
IT'S A NATIONAL RESEARCH
CONSORTIUM WHICH WAS FIRST
FUNDED IN 2017 TO DISCOVER AND
CHARACTERIZE THE RANGE OF
MOLECULAR TRANSDUCERS THAT UNDER
LIE THE AFFECT OF PHYSICAL
ACTIVITY IN HUMANS AND RATS OF
VARIOUS AGES.
THE PROGRAMS' GOAL IS TO
UNDERSTAND THE MOLECULAR CHANGES
DURING AND AFTER EXERCISE AND
ULTIMATELY TO ADVANCE THE
UNDERSTANDING OF HOW PHYSICAL
ACTIVITY IMPROVES AND PRESERVES
HEALTH.
RESULTS FROM PROFILING MOLECULES
IN WHOLE BLOOD, PLASMA AND 18
SOLID TISSUES FROM SIX-MONTH-OLD
RATS THAT UNDERWENT A TRAINING
PROGRAM HAVE BEEN PROVISIONALLY
ACCEPTED FOR PUBLICATION AND THE
PRE PRINT IS AVAILABLE NOW.
MY INSTITUTE DIRECTOR COLLEAGUES
AND I LEADING THE PROGRAM WROTE
A COMMENTARY THAT WILL BE
PUBLISHED WHEN THE PAPER IS
FORMALLY RELEASED.
WE'RE EXCITED ABOUT THIS
PROGRAM.
NOW MOVING ON TO THE IMPORTANT
TOPIC OF PAIN RESEARCH.
THE NIH HEAL INITIATIVE, THAT IS
THE HELPING TO END ADDICTION
LONG-TERM INITIATIVE IS AN
EFFORT TO SPEED SCIENTIFIC
SOLUTION TO STEM THE NATIONAL
OPIOID PUBLIC HEALTH CRISIS.
ALMOST EVERY NIH INSTITUTE AND
CENTER IS ACCELERATING RESEARCH
TO ADDRESS THIS PUBLIC HEALTH
EMERGENCY FROM ALL ANGLES.
AMONG THE EFFORTS NIAMS IS
LEADING THE BACPAC RESEARCH
PROGRAM WHICH IS A TRANSLATIONAL
PATIENT-CENTERED COLLABORATION
TO ADDRESS THE NEED FOR
EFFECTIVE AND PERSONALIZED
THERAPIES FOR CHRONIC LOW BACK
PAIN.
NIAMS IS ALSO LEADING THE
RESTORING JOINT HEALTH AND
FUNCTION TO REDUCE PAIN OR
REJOIN CONSORTIUM WHICH CONSISTS
OF RESEARCH TEAMS WORKING
TOGETHER TO MAP THE NETWORK OF
SENSORY NERVES THAT CONNECTS TO
TWO JOINTS THE TEMPOROMANDIBULAR
JOINT AND THE KNEE.
THE RESEARCH AIMS TO UNDERSTAND
HOW THE TYPES AND PATTERNS OF
SENSORY NERVE NETWORKS IN JOINTS
CHANGE WITH DISEASE AND WITH
AGING.
FURTHER, THE NATIONAL CENTER OF
COMPLIMENTARY AND INTEGRATIVE
HEALTH OR NCCIH ALSO LEADS AN
EFFORT THAT SEEKS TO DEVELOP
QUANTITATIVE IMAGING AND OTHER
RELEVANT BIOMARKERS OF
MYOFASCIAL TISSUES AND THE HEAL
INITIATIVE PRECISION HUMAN PAIN
NETWORK LED BY THE NATIONAL
INSTITUTE OF NEUROLOGICAL
DISORDERS AND STROKE AIMS TO USE
PRIMARY HUMAN TISSUES AND CELLS
TO BUILD COMPREHENSIVE DATA SETS
OF MOLECULAR SIGNATURES, CELL
TYPES AND CELL FUNCTIONED
PHENOTYPES OR SIGNATURES THAT
UNDER LIE HUMAN PAIN SIGNAL
TRANSDUCTION, TRANSMISSION AND
PROCESSING.
SO A CRITICAL PART OF HEAL IS
COLLECTING DATA THAT CAN BE
INTEGRATED ACROSS MODELS AND
ACROSS THE MAINS TO BETTER
UNDERSTAND CHRONIC PAIN.
SO WE'RE STARTING TO CREATE AN
ECO SYSTEM ADDRESSING THE
MULTIPLE DOMAINS ESSENTIAL TO
UNDERSTAND AND TREAT JOINT PAIN
FROM THE ENVIRONMENT, BEHAVIORAL
AND PSYCHOSOCIAL FACTORS TO
COMPLEX MOLECULAR INTERACTIONS.
AND NOW WITH COLLABORATIVE
EFFORTS AMONG HEAL PROGRAMS CAN
START TO MAKE SIGNIFICANT
ADVANCES AGAINST THE CHRONIC
PAIN THAT PLAIN OUR PATIENTS AND
SOCIETY.
AS WE WILL MOVE FORWARD
UNDERSTANDING PAIN REMAINS A
PRIORITY FOR NIAMS.
RELATED TO HEAL, I'D LIKE TO
ALSO HIGHLIGHT THE HEAL KIDS'
PAIN INITIATIVE.
KIDS STANDING FOR KNOWLEDGE,
INNOVATION AND DISCOVERY
STUDIES.
SO THE EUNICE KENNEDY SCHRIEVER
NATIONAL HEALTH OF HUMAN HEALTH
AND DEVELOPMENT ISSUED TWO
NOTICES OF FUNDING
OPPORTUNITIES.
ONE NOTICE IS FOR A PAIN
RESOURCE AND DATA CENTER AND THE
OTHER FOR SUPPORTING AN ACUTE
PAIN CLINICAL TRIALS PROGRAM.
NIAMS SIGNED ON TO BOTH AND
WE'RE VERY ENTHUSIASTIC ABOUT
THE OPPORTUNITY TO SUPPORT PAIN
RESEARCH IN INFANTS, CHILDREN
AND ADOLESCENTS WHICH IS A
COMMON ISSUE BUT OFTEN UNDER
RECOGNIZED IN CONSISTENTLY
ASSESSED OR MEASURED AND
INADEQUATELY TREAT.
WITHIN THE NIAMS EFFORT IT CAN
RELATE TO ACUTE ORTHOPEDIC PAIN
FROM COMMON INJURIES OR TO ACUTE
PAIN CAUSED BY DISEASE FLARES IN
CHILDREN WITH CHRONIC CONDITIONS
WITHIN OUR INITIATIVE AND THIS
UNDER SCORES NIAMS' COMMITMENT
TO BETTER UNDERSTANDING HEALTH
AND DISEASE IN DIVERSE
POPULATIONS AS SPECIFIED IN OUR
CURRENT STRATEGY PLAN.
ON THE FRONT, NIAMS PARTICIPATES
IN ANOTHER TRANS-NIH EFFORT LED
BY THE OFFICE OF RESEARCH ON
WOMEN'S HEALTH TO BETTER
UNDERSTAND CHRONIC CONDITIONS
UNDER STUDIED AMONG WOMEN AND/OR
DISPROPORTIONATELY AFFECT
POPULATIONS OF WITH UNDER
STUDIED AND UNDER REPRESENTED
AND UNDER REPORTED IN BIOMEDICAL
RESEARCH.
WE ALSO PUBLISHED TWO NOTICES OF
SPECIAL INTEREST THIS SUMMER.
THESE NOSIS ANNOUNCED OUR
INTEREST IN RECEIVING
ADMINISTRATIVE SUPPLEMENT TO
GRANT AND GRANT APPLICATIONS
THAT FOCUSSED ON SKIN RESEARCH
UNCOVER THE BASIS FOR SUBOPTIMAL
DIAGNOSES, INCREASED PREVALENCE
AND ELEVATED CLINICAL SEVERITY
AND TREATMENT RESPONSES IN BLACK
AMERICANS AND HISPANICS AND
LATINOS AND AMERICAN INDIANS AND
ALASKAN NATIVES AND ASIAN AND
ASIAN AMERICANS AND PACIFIC
ISLANDERS WITH SKIN DISEASES.
FURTHER, AS PART OF THE NIH-WIDE
UNITE INITIATIVE TO END
STRUCTURAL RACISM, THE NIH
ANNOUNCED ITS RESEARCH WITH
ACTIVITIES RELATED TO DIVERSITY
OR REWARD PROGRAM IN THIS PAST
MARCH.
REWARD PROVIDES SUPPORT FOR
SCIENTISTS MAKING A SIGNIFICANT
CONTRIBUTION TO DIVERSITY,
EQUITY, INCLUSION AND
ACCESSIBILITY IN THE RESEARCH
ENTERPRISE AND WHO HAVE NO
CURRENT RESEARCH GRANT FUNDING.
THIS FILLS AN IMPORTANT GAP.
APPLICANTS WILL RECEIVE THE RO1
MECHANISM FOR AND DEIA
ACTIVITIES IF SUCCESSFUL.
WITH THE NIAMS AS REPORTED IN
THE LAST DAY IN 2021 WE'RE
COMMITTED TO ELIMINATING
STRUCTURAL RACISM IN THE WO
WORKPLACE AND HEALTH CARE
SETTINGS.
FIRST THE STEPS WE'RE TAKING
WITHIN THE INSTITUTE.
WE CONTINUE TO FOCUS ON
EMPOWERING AND SUPPORTING OUR
STAFF WITH OPPORTUNITIES TO
SHARE AND LEARN ABOUT DEIA.
THIS YEAR WE PARTNERED WITH THE
NIH EQUITY, DIVERSITY AND
INCLUSION SESSIONS FOR INFORMAL
CONVERSATION ON DEIA WITH THE
NIH EQUITY, DIVERSITY AND
INCLUSION DIRECTOR.
THE SESSIONS OCCURRED BETWEEN
MAY AND JULY.
IN 2020 THE INSTITUTE
ESTABLISHED THE NIAMS CULTURE
COMMITTEE TO PROMOTE AN
ORGANIZATIONAL CLIMATE DESIGN BY
INCLUSIVITY, CIVILITY AND
DIVERSITY.
AMONG THE ACTIVITY THE COMMITTEE
ADVISES THE INSTITUTE'S
LEADERSHIP ON RECRUITING,
RETAINING AND PROMOTION
EMPLOYEES FROM UNDER REPRESENTED
BACKGROUNDS.
AND AS WE LOOK TO BRING PEOPLE
INTO THE NIAMS FOLD, WE'RE
SEEKING TO ENSURE A CIVIL
WORKPLACE ENVIRONMENT IN WHICH
WE EMPHASIZE WORKING TOGETHER
TOWARD A COMMON GOAL WITH
INTEGRITY, ACCOUNTABILITY AND
RESPECT FOR DIVERSITY OF
EXPERIENCES AND PERSPECTIVES.
SO TO THIS END THE NIAMS CULTURE
COMMITTEE HAS BEEN WORKING WITH
STAFF AND LEADERSHIP TO UPDATE
THE CORE VALUES DEVELOPED WITH
BROAD INPUT FROM STAFF AND
ROLLED OUT THIS MONTH.
THE NIAMS CORE VALUES UNDER
SCORE THE COMMITMENT TO
CULTIVATING A WORKPLACE THAT IS
RESPECT.
AND INCLUSIVE AND WILL INSPIRE
AND SUPPORT US AS WE ADVANCE
RESEARCH TO IMPROVE THE HEALTH
OF PEOPLE IN THE UNITED STATES.
THE NIAMS STRATEGIC PLAN FOR
FISCAL YEARS 2020 TO 2024
RECOGNIZING THE MISSION MAY
DISPROPORTIONATELY IMPACT
CERTAIN GROUPS AND LOOKING TO
ENHANCE WORKFORCE DIVERSITY IN
OUR MISSION AREAS.
THROUGH OUR SCIENCE
COMMUNICATION AND OUTREACH
BRANCH, NIAMS ENGAGES AND SERVES
AMERICANS FROM DIVERSE
BACKGROUNDS IN ADDITION THE
INSTITUTE ESTABLISHED A
DIVERSITY SCHOLARS PROGRAM TO
PROVIDE SUPPORT AND ENHANCE WITH
OUR SUPPLEMENT AWARDEES AND
MENTORS.
TO HELP US ADVANCE ALL THE
EFFORTS I'M DELIGHTED TO
ANNOUNCE NIAMS HAS NAMED
DR. ROBERT WALKER JR. AS THE
INSTITUTE'S FIRST CHIEF
DIVERSITY OFFICER.
UNTIL APRIL HE SERVED AS THE
DIRECTOR OF THE OFFICE FOR
STRATEGIC OPERATIONS WITHIN OUR
INTRAMURAL RESEARCH PROGRAM.
THERE HIS RESPONSIBLES INCLUDED
LEADING CAREER DEVELOPMENT FOR
THE TRAINING AWARDEES,
TECHNOLOGY TRANSFER, INNOVATION
DEVELOPMENT, SCIENTIFIC
COMMUNICATION, POLICY, INCLUSION
AND LOGISTICS AND IN HIS NEW
ROLE DR. WALKER WILL LEAD THE
DEVELOPMENT AND IMPLEMENTATION
OF RESEARCH, RESEARCH CAPACITY
BUILDING, TRAINING AND CAREER
DEVELOPMENT PROGRAMS TO SUPPORT
THE SCIENTIFIC MISSION OF NIAMS
AND ENHANCE SCIENTIFIC
DIVERSITY.
HE'LL PROVIDE RECOMMENDATIONS ON
SCIENTIFIC DIVERSITY GOALS AND
OBJECTIVES TO ENSURE DIVERSITY
IS CONSIDERED IN THE
DEVELOPMENT, FUNDING AND
ADMINISTRATION OF GRANT AWARDS,
COOPERATIVE AGREEMENTS AND
CONTRACTS AND IN ADDITION HE'LL
SERVE AS THE NIAMS LIAISON WITH
THE SCIENTIFIC COMMUNITY AND
PARTNER WITH THE NIH CHIEF
OFFICER FOR SCIENTIFIC WORKFORCE
DIVERSITY, THE OFFICE OF EQUITY,
DIVERSITY AND INCLUSION AND
CHIEF DIVERSITY OFFICERS FROM
OTHER NIH INSTITUTES AND
CENTERS.
FINALLY, I'D LIKE TO QUICKLY
UPDATE YOU ABOUT THE NIAMS
STRATEGIC PLANNING PROCESS AND
EXPECT YOU'LL HEAR MORE FROM
DR. BURROWS THE CHIEF OF OUR
PLANNING AND POLICY ANALYSIS
BRANCH THIS AFTERNOON.
I'M GRATEFUL TO THOSE WHO
VOLUNTEERED YOUR TIME TO ATTEND
THE THREE-DAY MEETING LAST WE
CAN OR WHO MADE THE EXTRA EFFORT
TO FIND A COLLEAGUE TO
PARTICIPATE IN YOUR PLACE.
RESEARCHERS AND STAFF ACROSS THE
NIH AND SEVERAL PATIENT
REPRESENTATIVES JOINED US TO
SHARE IDEAS ON CROSS-CUTTING
TOPICS THE INSTITUTE WILL PURSUE
THROUGH THE END OF THE DECADE.
IT WAS A FABULOUS EVENT FOR
THOSE THAT PARTICIPATED KNOW AND
I'M THRILLED WITH THE OUTCOME.
NOW, NIAMS STRATEGIC PLANS ARE A
PUBLIC STATEMENT OF THE
INSTITUTE'S INTEREST, A GUIDE
POST FOR THE SCIENTIFIC
COMMUNITY AND INTERNAL REFERENCE
DOCUMENT.
THERE ARE WE'RE MAKE CONCERTED
EFFORT TO CANVAS AND PROVIDE
OPPORTUNITIES TO PROVIDE INPUT
AND PARTICIPATING IN THE
INVITATION ONLY GENERATION
MEETING I REFERRED TO OR VIA
REQUEST FOR INFORMATION SUCH AS
THE ONE WE ISSUED LAST FALL.
THIS FALL THE INSTITUTE WILL
AGAIN ISSUE AN RFI TO GET
FURTHER INPUT ON A SUBSET OF
TOPICS THAT WERE IDENTIFIED BY
PARTICIPANTS AT THE STRATEGIC
PLANNING MEETING NEXT WEEK.
IN THIS WAY EVERYONE WILL HAVE A
CHANCE FOR THE VOICES TO BE
HEARD EVEN IF THEY DIDN'T ATTEND
THAT IN PERSON MEETING.
I SHOULD SAY THE IDEA GENERATION
MEETING.
IT WAS SO GOOD IT FELT IN
PERSON.
WE HOPE YOU'LL SPREAD THE WORD
ABOUT THE RFI WHEN IT'S
PUBLISHED.
SO BEFORE I CLOSE THOUGH I'D
LIKE TO DRAWER YOUR ATTENTION TO
A REQUEST FOR INFORMATION NIH
PUBLISHED WEEKS AGO IN RESPONSE
FROM THE ADVISORY COMMITTEE TO
THE DIRECTOR WORKING GROUP ON
DIVERSITY, NIH HAS DEVELOPED A
PROPOSED REVISED MISSION
STATEMENT AND SEEKING INPUT FROM
INTEREST GROUPS THROUGHOUT THE
SCIENTIFIC RESEARCH, ADVOCACY
AND CLINICAL PRACTICE COMMUNITY.
THOSE EMPLOYED BY NIH OR
INSTITUTIONS RECEIVING NIH
SUPPORT AND THE PUBLIC ON THE
PROPOSED STATEMENT.
ADDITIONAL INFORMATION ABOUT THE
EFFORT AND HOW TO PROVIDE INPUT
IS AVAILABLE ON THE NIH WEBSITE
AND I ENCOURAGE ALL OF YOU TO
REVIEW THE PROPOSAL, SHARE IT
WITH YOUR COMMUNITIES AND
CONSIDER PROVIDING YOUR INPUT.
FINALLY, FOR THOSE INTERESTED IN
LEARNING MORE ABOUT OUR
PROGRAMS, PLEASE VISIT THE NIAMS
WEBSITE.
YOU CAN ALSO FIND US ON
FACEBOOK, LINKEDIN AND YOUTUBE
AND TO LEARN MORE ABOUT OUR
FUNDING PROGRAMS SUBSCRIBE TO
OUR NEWSLETTER AND FOLLOW OUR
FORMERLY KNOWN AS TWITTER
ACCOUNT.
THAT CONCLUDES MY REMARKS.
I'M HAPPY TO TAKE ANY QUESTIONS
YOU MIGHT HAVE AT THIS TIME.
OKAY.
I'M AFRAID I CAN'T SEE NAME
TAGS.
>> MY QUESTION IS THERE'S BEEN
SUCH PUSHBACK ON DEI IN THE
FEDERAL GOVERNMENT.
SORRY.
THERE'S BEEN SUCH A PUSHBACK ON
DEI IN THE FEDERAL GOVERNMENT
GOVERNMENT PARTICULARLY IN THE
STATE OF FLORIDA.
SO HAVE YOU RUN INTO OR GETTING
STARTED ON YOUR NEW DIVISION
WITH DEI, DO YOU THINK YOU'LL
RUN INTO ANY PUSHBACK ON THAT
BECAUSE IT SEEMS LIKE THEY'RE
TRYING TO GET RID OF DEI IN A
LOT OF THE DIFFERENT
CORPORATIONS AND ORGANIZATIONS.
>> RIGHT.
WELL, WE AT THE NIH ARE UNIFIED
IN OUR SUPPORT OF DIVERSITY,
EQUITY, INCLUSION AND
ACCESSIBILITY ISSUES AND
CONCERNS AND PROGRAMS SO WE ARE
MOVING AHEAD AND DOING
EVERYTHING THAT WE CAN.
WE FEEL MORALLY OBLIGED TO MAKE
PROGRESS IN THE AREA AND IT'S A
MAJOR INITIATIVE AND WE'RE BEING
MINDFUL OF CONSIDERATIONS SUCH
AS GUIDANCE FROM THE GENERAL
COUNSEL AND OTHER GUIDANCE WE
MAY BECOME AWARE OF BUT WE'RE
COMMITTED TO MAKING PROGRESS IN
THIS AREA AND ACTUALLY OUR
ACTING NIH DIRECTOR JUST WALKED
IN AND I APPLAUD HIS DEEP
COMMITMENT TO THIS AREA.
YOU'LL HEAR FROM HIM IN A COUPLE
MINUTES BUT WE COULD HAVE NOT
ASKED FOR A MORE SUPPORTED AND
COMMITTED LEADER ON THAT FRONT.
I SHARE YOUR CONCERN BUT I WANT
TO REASSURE YOU, WE'RE REALLY
COMMITTED TO PROGRESS IN THIS
AREA.
OTHER QUESTIONS?
I'M AVAILABLE ALL DAY FOR THOSE
WHO WANT TO CATCH ME IN PRIVATE.
HAPPY TO TAKE QUESTIONS AT THIS
TIME.
WE HAVE PLENTY OF TIME.
>> THANK YOU, DR. CRISWELL.
GREAT TALK.
WE HAVE A LOT OF RARE DISEASES
IN THE ROOM HERE TODAY.
I KNOW YOU MENTIONED A FEW OF
THE RARE DISEASES GETTING
SPECIFIC FOCUS AND ATTENTION BUT
A LOT OF THE FOLKS ARE ALWAYS
ASKING QUESTIONS ABOUT HOW DO
THEY BETTER ENGAGE WITH THE NIH
AND WOULD LOVE TO HEAR GUIDANCE
FROM YOU ON THAT.
>> RARE DISEASES ARE EXTREMELY
IMPORTANT AND NIAMS HAS A
HISTORY OF SUPPORTING RARE
DISEASE AND I'M PROUD OF THAT
AND OTHER INSTITUTES AND CENTERS
RECOGNIZE THE IMPORTANT AND WE
HAVE A LOT TO LEARN IN DRILLING
DOWN TO THE ROOT CAUSES OF RARE
DISEASES BECAUSE THEY CANNOT
ONLY PROVIDE SOLUTIONS FOR THOSE
WHO SUFFER WITH THOSE CONDITIONS
BUT PROVIDE INSIGHT INTO OTHER
MORE COMMON CONDITIONS.
SO I THINK THERE IS ABSOLUTELY
EVERY REASON TO BE PURSUING
EFFORTS IN RARE DISEASE AS WELL
AS COMMON DISEASE.
INCREASINGLY, WE RECOGNIZE THE
DEFINITIONS THAT WE HAVE
DEVELOPED FOR DISEASES AND
CONDITIONS ARE REALLY OFFICIAL.
THEY DON'T REFLECT THE
UNDERLYING BIOLOGY AND IN SOME
RESPECTS THEY POSE SOME
CONSTRAINT.
WE'RE TRYING THROUGH THE
ACCELERATED MEDICINES
PARTNERSHIP AND OTHER PROGRAMS
RECOGNIZE WE NEED TO MAKE SURE
THAT THE DISEASE DEFINITIONS AND
HOW WE'RE ORGANIZED IN OUR
STRUCTURES DON'T INTERFERE WITH
UNDERSTANDING THE BIOLOGY OF THE
DISEASE.
I'M EXCITE ABOUT A NEW EFFORT I
DIDN'T MENTION HAPPENING IT'S
CALLED SYSTEMS BIOLOGY OF
INFLAMMATION AND RECOGNIZES THE
FACT THAT IF WE CAN INTEGRATE
KNOWLEDGE AND DATA ACROSS
MULTIPLE DIFFERENT TYPES OF
DISEASE, WE'LL DISCOVER
CONNECTIONS WE HADN'T
APPRECIATED AND WE MAY NOT EVER
DISCOVER IF WE CONTINUE TO FOCUS
ON THE DISEASE AND CONDITION
DEFINITIONS WE CURRENTLY HAVE.
SO ANYWAY, I HOPE THAT HELPS
ADDRESS SOME OF YOUR CONCERNS
THERE.
ANY OTHER QUESTIONS AT THIS
POINT?
LOOKS LIKE LEE HAS A QUESTION.
WE'RE ALL GATHERED HERE, 40 PLUS
ADVOCACY ORGANIZATIONS HERE.
WHAT DO YOU THINK WOULD BE THE
BEST WAY FOR US TO SUPPORT NIAMS
GOING FORWARD?
>> YOU CAN HELP US TREMENDOUSLY
BY MAKING MORE VISIBLE THE
IMPORTANCE OF THE CONDITIONS
WITHIN OUR MISSION AREAS, THE
CHALLENGES THAT INDIVIDUALS AND
THEIR FAMILIES, THE IMPACT IN
TERMS OF QUALITY OF LIFE, LENGTH
OF LIFE, THE FINANCIAL IMPACT.
WE'RE SOMETIMES CHALLENGED TO BE
AS VISIBLE AS WE THINK WE SHOULD
BECAUSE MANY OF THE DISEASES
DON'T RESULT IN THE END OF LIFE.
AND THEY IMPACT LIFE.
SOMETIMES THE IMPORTANCE OF
THOSE CONDITIONS IS NOT
ADEQUATELY APPRECIATED.
I WOULD ALSO ENCOURAGE YOU TO
WORK TOGETHER SO THAT INSTEAD OF
SPEAKING 90 PLUS INDIVIDUAL
ORGANIZATIONS YOU'RE SPEAKING AS
THE COALITION.
IF I WAS TRYING TO UNDERSTAND
THE LANDSCAPE IT WOULD BE
HELPFUL TO ME SO I ENCOURAGE YOU
TO THINK WITH THE ISSUES AND
CONCERNS YOU SHARE AND HOW YOU
CAN WORK TOGETHER TO REPRESENT
OUR MISSION AREAS.
I HOPE THAT THAT SORT OF CONCEPT
IS SOMETHING YOU CAN THINK
ABOUT.
I THINK THAT WOULD BE REALLY
HELPFUL.
OTHER QUESTIONS AT THIS POINT?
WELL, WE'RE A LITTLE HEAD OF
SCHEDULE.
I SEE DR. TABAK SO MAYBE WE'LL
MOVE AHEAD.
THANK YOU FOR YOUR ENGAGEMENT ON
MY STATE OF THE INSTITUTE AND
I'LL INTRODUCE DR. TABAK THE
DIRECTOR OF THE NATIONAL
INSTITUTES OF HEALTH WHO
OFFICIALLY TOOK OFFICE DECEMBER
20, 2021.
NEEDLESS TO SAY HIS LIST OF
ACCOMPLISHMENTS AND SERVICE TO
THE NIH IS LONG BUT I'D LIKE TO
SHARE SOME HIGHLIGHTS.
DR. TABAK HAS SERVED AS THE
PRINCIPLE DEPUTY DIRECTOR AND
DEPUTY ETHICS COUNSELOR SINCE
2010 AND SERVED THERE'S ACTING
DIRECTOR OF NIH AND THE DIRECTOR
OF CRANIOFACIAL RESEARCH FRAND S
THE SENIOR ASSOCIATE DEAN FOR
RESEARCH AND PROFESSOR OF
DENTISTRY AND BIO CHEMISTRY AT
THE SCHOOL OF DENTISTRY AT THE
SCHOOL OF RODCHESTER, NEW YORK
AND A MERIT AWARD RECIPIENT
EXTENDING RESEARCH IN TIME.
THAT MEANS HE WAS A FANTASTIC
NIH AWARD RECIPIENT AND HIS
MAJOR RESEARCH FOCUS HAS BEEN ON
THE STRUCTURE AND FUNCTION OF
GLYCOPROTEINS.
HE CONTINUES TO WORK ON HIS AREA
MAINTAINING AN ACTIVE RESEARCH
LABORATORY WITHIN THE NIH
PROGRAM IN ADDITION TO HIS MANY
ADMINISTRATIVE DUTIES AND IS
ELECTED TO THE NATIONAL ACADEMY
OF MEDICINE AF THE NATIONAL
ACADEMIES AND RECEIVED HIS
UNDERGRADUATE FROM COLLEGE OF
NEW YORK AND DDSD FROM COLUMBIA
UNIVERSITY AND Ph.D. FROM THE
UNIVERSITY OF BUFFALO.
DR. TABAK, THANK YOU FOR BEING
HERE TO WELCOME PARTICIPANTS TO
THE 2023 NIAMS COALITION
OUTREACH AND EDUCATION DAY AND I
KNOW THAT YOU WERE ABLE TO
WELCOME US VIA VIDEO IN 2019 AND
NOW THANK YOU SO MUCH FOR BEING
WITH US.
>> THANK YOU FOR THAT ELABORATE
INTRODUCTION.
SO IT'S GREAT TO SEE ALL OF YOU
HERE IN PERSON.
I ACTUALLY SEE A COUPLE OF
FAMILIAR FACES FROM MY TIME AT
NIDCR.
THERE WAS SOME OVERLAP IN OUR
MISSION.
I WANTED TO DO THIS BECAUSE
YOU'RE WHY WE HAVE NIH.
IT'S JUST WONDERFUL TO HAVE SO
MANY OF YOU HERE IN ONE ROOM
TOGETHER.
IT'S BEEN ALL TOO LONG ACTUALLY.
SO I'VE BEEN ACTING DIRECTOR NOW
SINCE DECEMBER OF '21.
SO NEXT MONTH IS 22 MONTHS AND
COUNTING THOUGH THERE IS HOPE ON
THE HORIZON AND WE'RE VERY MUCH
LOOKING FORWARD TO
DR. BERTAGNOLLI'S SPEEDY
CONFIRMATION TO WHICH WE SAY
AMEN.
BUT I'VE BEEN AROUND HERE FOR A
WHILE.
SOME WOULD THINK I CAN'T HOLD A
JOB ANY PLACE BECAUSE OF THE
LITANY OF THINGS I'VE BEEN DOING
AND THROUGH ALL THOSE ACTIVITIES
ONE THING COMES BACK OVER AND
OVER AGAIN.
I'LL TALK INTO THIS THING.
THIS WORKS.
LET'S ADAPT.
IT WILL BE OKAY.
I'VE LEARNED OVER AND OVER AGAIN
HOW IMPORTANT YOUR VOICES ARE
AND I APPRECIATE THAT YOU EACH
REPRESENT YOUR OWN SPECIFIC AREA
OF INTEREST AND THAT'S GREAT BUT
AS DR. CRISWELL SAID, SOMETIMES
BANDING TOGETHER IS MUCH MORE
POWERFUL AND IF YOU APPROACH
MEMBER OF CONGRESS AND TALK
ABOUT 90 DIFFERENT ORGANIZATIONS
REPRESENTING HOWEVER, MANY
THOUSANDS AND THOUSANDS OF
PEOPLE, TENS OF THOUSANDS OF
PEOPLE, THAT HAS TREMENDOUS
EFFECT.
WE HAVE WORKED CLOSELY WITH
ORGANIZATIONS SUCH AS THE ONES
YOU REPRESENT FOR MANY YEARS.
I CAN TELL YOU AS AN INSTITUTE
DIRECTOR IT INFLUENCED
DRAMATICALLY HOW WE POSITIONED
OURSELVES, HOW WE REORGANIZED
PRIORITIES TO BE MORE PATIENT
CENTRIC.
AND I KNOW NIAMS HAS A HISTORY
OF THAT AND THE LATE STEVE KATZ
AND I WERE COLLEAGUES AND WE
USED TO TALK ABOUT THAT A LOT
AND DR. CRISWELL HAS CONTINUED
AND BUILT UPON THAT IN A VERY
REAL WAY.
WE'RE REALLY TRYING TO BE MUCH
MORE RESPONSIVE TO PATIENT
GROUPS.
THE SPECIFIC ISSUE OF RARE
DISEASES BROUGHT UP AS A WALKED
IN, CONGRESS IS ALWAYS ASKING ME
ABOUT RARE DISEASES.
THEY TAKE OUT THEIR CALCULATORS
AND I DON'T KNOW HOW YOU
CALCULATE A PERSON'S LIFE BUT IF
YOU HAVE THE DISEASE IT'S NOT SO
RARE.
THAT USUALLY GETS THEM TO THINK
ABOUT THIS IN A SOMEWHAT
DIFFERENT WAY.
I DO THINK YOU HAVE COMING UP A
WHOLE SERIES OF IMPORTANT
VISITORS TO YOUR GROUP YOU HAVE
SPEAKERS FROM THE FOUNDATION OF
NIH.
DR. CRISWELL MENTIONED SOME OF
THE AMP PROGRAMS, ACCELERATING
MEDICINE PARTNERS PROGRAM WHICH
NIMS HAS TAKEN A LEADERSHIP ROLE
AND BOB CARTER MADE AN AMAZING
ROLE AND I THANK HIM FOR THAT.
WHAT THE AMP PROGRAMS HAVE DONE
AND HE CAN TELL YOU MORE THAN I
CAN, HAVE FORGED A RELATIONSHIP
WITH NON FOR PROFITS, PATIENT
GROUPS AND THE NIH AND THEY'RE
IN AREAS NEAR AND DEAR TO MANY
OF YOUR INTERESTS.
YOUR ALSO GOING TO HEAR FROM OUR
COLLEAGUES AT ARPA-H THE NEWEST
ENTITY WITHIN THE DEPARTMENT OF
HEALTH AND HUMAN SERVICES AND
THEY'LL TELL YOU WITH THEIR
CUTTING-EDGE IDEAS THEY'RE
IMPLEMENTING.
THERE'S A POSTER SESSION AND A
TIME TO SHARE BEST PRACTICES
FROM EACH OF YOUR ORGANIZATIONS
AND MOST IMPORTANTLY TIME WITH
THE STAFF.
MAKE GOOD USE OF THAT TIME.
THAT'S THE WHOLE POINT.
WE WANT TO INFORMALLY LEARN FROM
ONE ANOTHER AND THE TIME WITH
STAFF WILL GO AN ALONG WAY IN
DOING THAT.
WHEN YOU LEAVE THE MEETING, WHAT
I HOPE YOU LEAVE WITH IS AN
INCREASED APPRECIATION FOR NIMS
AND THE NIH IN GENERAL THAT YOU
HAVE EXPANDED YOUR NETWORK OF
COLLEAGUES SO IF YOU'RE
CURRENTLY SEATED WITH PEOPLE YOU
KNOW, THE BREAK, SIT DOWN WITH
PEOPLE YOU DON'T KNOW.
IT'S IMPORTANT BECAUSE BY THE
TIME YOU LEAVE YOU SHOULD KNOW
EVERYBODY IN THE ROOM.
YOU'LL COME AWAY WITH RESOURCES
NIMS ARE ABLE TO PROVIDE AND
ADDITIONAL ONES YOU'LL LEARN
ABOUT FROM ARPA-H AND MAYBE
THINGS FROM THE FOUNDATION.
ADD TO YOUR TOOL KIDS.
I DO HAVE A QUICK REQUEST.
I CAN'T COME WITHOUT A REQUEST,
RIGHT.
SO WE'RE IN THE PROCESS OF
REWORDING OUR MISSION STATEMENT.
I HEARD A LITTLE BIT ABOUT OUR
COMMITMENT TO ENSURING THAT THE
"A" IN DEIA ACTIVITIES IS WELL
REPRESENTED AND ADDRESSED.
WE PUT OUT A REQUEST FOR
INFORMATION ABOUT THE MISSION
STATEMENT AND WE REALLY
ENCOURAGE ALL OF TO YOU RESPOND
TO THE RFI, REQUEST FOR
INFORMATION FROM YOUR RESPECTIVE
VIEW POINTS.
I'M SURE IF I SPEAK TO THREE OF
YOU THERE'LL BE FIVE VIEW POINTS
AND THAT'S FINE, THAT'S WHAT WE
WANT TO HEAR.
BECAUSE THESE CHANGES -- MISSION
STATEMENTS MATTER.
MAY THINK, OH, WHAT'S THE BIG
DEAL.
MISSION STATEMENTS MATTER.
AND WE REALLY FEEL THE INPUT
FROM ORGANIZATIONS SUCH AS THE
ONES YOU ALL REPRESENT ARE
CRITICALLY IMPORTANT SO WE CAN
TRY AND GET IT RIGHT.
WILL EVERYBODY AGREE?
NO.
BUT WE WANT TO GET AS CLOSE TO
CONSENSUS AS IS POSSIBLE BECAUSE
NOT ONLY WHAT WE DO BUT WHAT WE
STAY HAS AN AFFECT ON PEOPLE.
THAT MISSION STATEMENT CAN HAVE
AN EFFECT ON PEOPLE AND THAT'S
WHY WE'RE IN THE SPACE WE ARE
NOW CONSIDERING ITS REVISION.
I HOPE TODAY'S MEETING WILL
REINFORCE THE IMPORTANCE OF THE
PARTNERSHIP WE HAVE.
THE FACT THAT YOU TOOK THE TIME
AND TROUBLE TO BE HERE SPEAKS
VOLUMES ABOUT YOUR COMMITMENT TO
NIH AND NIMS AND TO ONE ANOTHER
SUPPORTING EACH OTHER.
I REALLY THINK THIS IS THE
PRICELESS ASSET YOU PROVIDE TO
US.
AND REALLY JUST WANT TO COMMEND
ALL OF YOU FOR THE GREAT WORK
THAT YOU DO.
AND SO I DON'T KNOW, LINDSEY, IF
YOU WANTED ME TO TAKE ANY QUICK
QUESTIONS.
IF THERE'S ANY QUICK QUESTIONS
PEOPLE HAVE, I'M HAPPY TO TRY
AND ANSWER THEM BUT IF NOT WE'LL
TURN IT BACK TO DR. CRISWELL.
>> HERE'S A RARE OPPORTUNITY.
>> YES.
>> THANK YOU FOR SHARING WITH US
TODAY.
WE WERE HEARING FROM
DR. CRISWELL AND WE WERE
CHATTING ABOUT THE COMMONALITY
ACROSS OUR DISEASES AND HOW DO
YOU WORK ACROSS SO MANY
INSTITUTES AND CENTERS?
>> THAT'S AN IMPORTANT QUESTION.
ONE THAT WE SORT OF STRUGGLE
WITH EACH AND EVERY DAY.
SO MUCH OF THIS IS DONE AT THE
INSTITUTE LEVEL.
STAFF MEMBERS INTERACTING WITH
STAFF MEMBERS TO KEEP EACH OTHER
APPRISED OF WHAT'S ACTUALLY
GOING ON AT THE GROUND LEVEL.
WE HAVE AN INSTITUTE AND CENTERS
DIRECTORS MEETING USUALLY EVERY
WEEK WHERE PEOPLE SHARE WHAT IT
IS THEY HAVE COMING UP TO TRY
AND ENTICE PEOPLE TO WORK
TOGETHER ON CERTAIN THINGS.
WE HAVE FORMAL PLANNING ACROSS
THE AGENCY THROUGH A DIVISION
WITHIN THE OFFICE OF THE
DIRECTOR THE ACRONYM IS DPCPSI.
IT'S SOMETHING ABOUT STRATEGIC
PLANNING AND COORDINATION AND SO
FORTH.
ONLY THE CONGRESS COULD -- BUT
THAT ALLOWS FOR A MORE FORMAL
LANDSCAPE ANALYSIS, IF YOU WILL.
AND WE HAVE MANY TOOLS NOW THAT
ALLOW US TO INTER -- INTERROGATE
AND LOOK FOR SYNERGY AND LOOK
FOR GAPS AND I THINK WHEN YOU
TAKE ALL THOSE THINGS TOGETHER,
THAT'S HOW WE GO FORWARD AND TRY
TO INTEGRATE.
WE'RE A DECENTRALIZED
ORGANIZATION BUT THE LEADERSHIP
AT THE LEADERSHIP LEVEL OF
INSTITUTE AND CENTER DIRECTORS
THERE IS ALMOST UNANIMITY WITH
COHERENCE AND APPROACH TO
INTEGRATE AS MUCH AS POSSIBLE.
WE SAW THIS IN A VERY
SIGNIFICANT WAY DURING THE
PANDEMIC.
SOME OF THE THINGS DURING THE
PANDEMIC HAVE CARRIED OVER IN IF
WE WORK TOGETHER MAYBE WE CAN
ENGAGE COMMUNITIES IN A ROBUST
WAY.
WE'RE CONTINUING THOSE TYPES OF
ACTIVITIES.
I WISH THERE WAS ONE SINGLE
THING I COULD POINT TO BUT I
HAVE TO POINT TO A SERIES OF
THINGS TO ALLOW US TO DO THIS IN
AN OPTIMAL WAY.
NOW THAT WE HAVE ARPA-H, WE HAVE
TO ADD THAT INTO THE MIX AS WELL
BECAUSE THE FUNDAMENTAL QUESTION
THAT THEY ASK THAT WE ASK AND
CONGRESS ASKED IS ARE YOU DOING
SOMETHING NEW?
ARE YOU DOING PURPOSE LY
AUGMENTING WHAT IS BEING DONE.
YEAR INVOKING THE APPROACH TO --
WE'RE INVOKING THE APPROACHES TO
NAVIGATE THAT AS WELL.
>> HI, I'M OLIVIA CASEY WITH THE
AUTOIMMUNE ASSOCIATION.
HOW DO YOU SEE THE OADR
INTERACTING AND FUNCTIONING --
>> HOW DO I SEE?
>> THE OADR, OFFICE OF
AUTOIMMUNE DISEASE RESEARCH.
>> THIS IS THE NEWEST MEMBER OF
THE NIH FAMILY AND IT'S STILL
NASCENT.
I CAN ONLY DRAW UPON HOW OTHER
SUCH OFFICES AND PROGRAMS WORK
AND IT'S MOSTLY CONVENING POWER.
DON'T MINIMIZE THAT BECAUSE WHEN
YOU CONVENE, TYPICALLY PROGRAM
OFFICERS COME WITH THEIR
CHECKBOOKS.
THEY'RE LOOKING TO LEVERAGE
THEIR ACTIVITY WITH SOMEBODY
ELSE BECAUSE IF THEY BRING A
DOLLAR AND YOU BRING A DOLLAR,
NOW YOU HAVE TWO DOLLARS TO
SPEND TO ATTACK THE PROBLEM.
INITIALLY IT WILL BE LARGELY
CONVENING BUT FROM THAT I'M SURE
WILL COME SYNERGIES IN TERMS OF
JOINT FUNDING, IN TERMS OF NEW
PROGRAMS THAT REQUIRE MULTIPLE
INSTITUTES AND CENTER TO
PARTICIPATE.
AND DR. CRISWELL MENTION THE
WHOLE IDEA OF INFLAMMATION AS
SORT OF A COMMON THEME ACROSS SO
MANY DISEASES AND CONDITIONS.
VIRTUALLY EVERY INSTITUTE AND
CENTER SHOULD BE CONCERNED ABOUT
INFLAMMATION AT SOME LEVEL BUT
MANY HAVE ALWAYS -- NO, THAT'S
NIAID OR THAT'S NIMS OR WHO ARE
THEY POINT TO WHEN IN FACT THEY
SHOULD BE DOING THAT.
HAVING THAT CONVENING POWER AT
LEAST INITIALLY IS VERY CRUCIAL
BUT STAY TUNED BECAUSE IT REALLY
IS A NASCENT ORGANIZATION.
>> WE HEARD A LOT TODAY AND WHAT
IS THE MOST HELPFUL FOR
[INAUDIBLE]
>> I THINK MEETINGS LIKE THIS
ONE IS IMPORTANT BECAUSE THIS IS
WHAT CATALYZES US INTO ACTION.
YOU HAVE 90 DIFFERENT
ORGANIZATIONS REPRESENTED HERE.
THAT HOLDS OUR FEET TO THE FIRE,
IF YOU WILL.
IT HOLDS US ACCOUNTABLE.
WHAT WE ALSO LEARNED AND THIS
WAS PARTICULARLY TRUE DURING THE
PANDEMIC BUT IT'S CERTAINLY
SPILLED OVER INTO OTHER THINGS
UNLESS WE ENGAGE WITH PATIENTS
DIRECTLY AND WITH PEOPLE WITH
LIVED EXPERIENCE THE ANSWER IS
OVER HERE AND WE'RE OVER HERE
AND WE WANT TO MEET IN THE
MIDDLE.
I THINK THE CONTINUED EMPHASIS
BY ORGANIZATIONS SUCH AS
YOURSELVES AND SUPPORT FROM THE
CONGRESS FROM WHICH WE DERIVE
OUR FUNDING AND THE CONTINUING
UNDERSTANDING, THE EVOLUTION OF
SCIENTIFIC THOUGHT BIOMEDICAL
RESEARCH THOUGHT THAT YOU GOT TO
GET THE PATIENTS INVOLVED THAT
ARE IN A SERIOUS WAY.
I'M INTIMATELY INVOLVED WITH
SOMETHING CALLED THE HEAL
INITIATIVE WHICH I KNOW SOME OF
YOU ARE FAMILIAR WITH RELATED TO
PAIN AND OPIOID USE DISORDER.
I HAVE TO TELL YOU, WITHOUT
ENGAGING THE PATIENTS AND THOSE
WITH LIVED EXPERIENCES WE'D BE
IN A VERY DIFFERENT PLACE.
THE SAME FOR DISEASES AND
CONDITIONS EACH OF YOU
REPRESENT.
AND IT'S THE SAME THEME OF
COURSE.
I WILL TELL YOU DR. BERTAGNOLLI
HAS CLEARLY IS A VERY
PATIENT-CENTRIC INDIVIDUAL WITH
ALL THE WORK THAT SHE'S DONE IN
CANCER AS I PROJECT TO THE
FUTURE HOPEFULLY WITH A
CONFIRMATION WE'LL PROCEED IN A
VERY ROBUST WAY DOWN THAT PATH.
>> HI.
KAREN BALL WITH THE STURJ
FOUNDATION ONE OF THE RARE
DISEASES YOU MENTIONED.
I WAS HAPPY TO HEAR YOU TALK
ABOUT INFLAMMATION.
JUST WANTED TO THANK YOU AND
DR. CRISWELL AND DR. CARTER
BECAUSE FOR THE LAST 36 YEARS
THE MEETING OF THE DIVERSE
ORGANIZATIONS HAS DRIVEN OUR
INQUISITIVE UNDERSTANDING OF THE
FOUNDATION AND THE DISEASE.
SO MULTI INSTITUTES WE INTERACT
WITH AND NOW WE'RE BURGEONING ON
MORE CLINICAL TRIALS SINCE
DISCOVERING THE PPG AND THAT'S
OUR ONLY HOPE OF GETTING A LEG
UP.
ARE YOU GOING CONTINUE THAT OR
EXPAND IT?
>> WE'RE TALKING ABOUT HOW WE
MIGHT EXPAND THAT.
OBVIOUSLY IN THE SPACE OF RARE
DISEASE TRIALS TAKE ON THEIR OWN
COMPLEXITIES BECAUSE OF NUMBERS.
AND SO YOU NEED TO CONTEMPLATE
UNIQUE TRIAL DESIGN AND SO FO
FORTH.
THAT'S ALL THE MORE REASON TO
STRENGTHEN THE PPP APPROACH
BECAUSE UNLESS YOU'RE ABLE TO
ENGAGE ALL THE PATIENTS
SOMETIMES AND UNLESS YOU'RE ABLE
TO PARTNER WITH INDUSTRY UP
FRONT, IT DOES NOT MAKE FOR THE
OPTIMAL WAY FORWARD.
WE ARE TALKING ABOUT HOW WE CAN
STRENGTHEN THAT.
FOUNDATION AND YOU'LL HEAR FROM
THAT IN A BIT.
THEY'VE BEEN AN AMAZING PARTNER
AND I CAN'T SAY ENOUGH ABOUT
THEM THREE THE AMP SERIES OF
PROGRAMS AND OTHERS.
PLEASE CONTINUE TO ADVOCATE FOR
THAT.
AND KNOW THAT THE NIH LEADERSHIP
IS VERY MUCH SUPPORTIVE.
OKAY.
WITH THAT, THANK YOU SO MUCH
BECAUSE I SEE MY COLLEAGUE
DR. GERBERDING IN THE BACK
THERE.
>> THANK YOU SO MUCH, DR. TABAK.
I'M GLAD HE WAS ABLE TO TAKE
QUESTIONS.
IN THE NEXT PORTION WE'LL HEAR
FROM THE CURRENT NIAMS COALITION
CO-CHAIRS, TRISHA AND LEE
PROVIDING AN OVERVIEW OF
ACCOMPLISHMENTS AND AT EACH
MEETING WE TAKE A MOMENT TO
RECOGNIZE THE FORMER AND
OUTGOING CO-CHAIRS FOR THEIR
DEDICATION AND LEADERSHIP OF THE
NIAMS COALITION.
I'D FIRST LIKE TO RECOGNIZE MS.
MARCIOLLO CO-CHAIR FROM 2020 TO
2021.
WE HAVE A CERTIFICATE FOR HER.
I HOPE SHE'S HERE.
SO NEXT, I'D LIKE TO EXTEND OUR
APPRECIATION TO MS. TRISHA
SHIVAS AS THE SPENDS THE NEXT
MONTH WRAPPING UP HER SERVICE AS
CO-CHAIR.
WE ALSO HAVE A CERTIFICATE FOR
YOU.
OKAY.
SO WE LOOK FORWARD TO YOUR
CONTINUED PARTICIPATION AND
COALITION ACTIVITIES OVER THE
COMING YEARS.
SO I THINK WE'RE GOING TO HAVE A
PHOTO WITH CO-CHAIRS NOW.
WE'LL NOW TURN THINGS OVER TO
TRISHA AND LEE TO PROVIDE AN
OVERVIEW OF THE NIAMS ACTIVITIES
AND ACCOMPLISHMENTS.
LOOKING FORWARD TO IT.
>> SO WE WANTED TO KICK IT OFF
WITH SOMETHING A LITTLE FIN.
WE DON'T ALWAYS WANT THE
CELEBRITIES TO HAVE ALL THE FUN
WITH SELFIES AND WANTED TO DID A
GIGANTIC NIAMS' SELFIE AND SHOW
HOW INCREDIBLE IT IS ALL THE
PEOPLE IN THE ROOM AND LEE WILL
TAKE HIS BEST SHOT AT CAPTURING
EVERYBODY.
PUT YOUR BEST SMILES ON.
>> I'M REALLY BAD AT THIS.
THIS IS INDULGING US A LITTLE
BIT.
>> WE'RE GOING TO POST THAT AS
NOT OUR FORMAL PICTURE BUT A
MORE FUN.
>> I'M GOING APOLOGIZE IN
ADVANCE.
NEVER LET A BOOMER TAKE A
SELFIE.
>> WE'RE SO EXCITED TO HAVE YOU
HERE AND WANT TO WELCOME YOU
AGAIN.
THIS IS GOING TO BE A CHOCK-FULL
CONVERSATION AND ALSO AN
OPPORTUNITY FOR EVERYONE TO GET
TO KNOW ONE ANOTHER AND SHARE
BECAUSE ONE OF THE THINGS WE
HEARD YEAR AND YEAR FROM
EVALUATIONS IS HOW IMPORTANT IT
IS THE CONVERSATIONS YOU ARE ALL
HAVING WITH EACH OTHER.
WE ENCOURAGE YOU TO TAKE UP
OPPORTUNITIES DURING LUNCH, FIND
SOMEONE TO HAVE LUNCH WITH
DURING THE CONVERSATIONS AND GET
TO KNOW EACH OTHER.
>> AS YOU CAN SEE HERE, THIS IS
FROM 2019 THE LAST TIME THAT WE
MET IN PERSON.
THERE'S SOME OF YOU THAT ARE IN
THE ROOM TODAY THAT WERE IN THE
PICTURE AND FOR ALL OF US,
THERE'S SO MUCH THAT'S HAPPENED
IN THE LAST FOUR YEARS.
I THINK IT CAUSES US ALL TO BE
REFLECTIVE OF WHAT WE'VE GONE
THROUGH AND HOW THE WORLD'S
LITERALLY CHANGED IN SO MANY
WAYS.
I LIKE TO LOOK AT THIS AS BEING
A POSITIVE EFFECT.
WE'VE NOW ARE IN THE ASPECT OF
TRANSFORMATION.
THAT'S PART OF WHAT WE'RE TRYING
TO DO HERE WITH THE COALITION.
AND WHY WE'RE ALL HERE TODAY TO
LOOK AT WAYS TO TRANSFORM WHAT
WE'RE DOING AS AN ORGANIZATION
AND COALITION TO MOVE IT FORWARD
TO WE'RE MORE PRODUCTIVE IN
SUPPORTING NIAMS AND OUR
ORGANIZATIONS.
I WANT TO THANK OUR COALITION
STEERING COMMITTEE.
I WON'T NAME ALL THE PEOPLE HERE
BUT PLEASE TAKE NOTE OF ALL OF
THOSE NAMES AND AS YOU GO
THROUGH THE DAY TAKE TIME ALSO
TO THANK EACH AND EVERY ONE OF
THESE INDIVIDUALS FOR THEIR
SUPPORT AND FOR HELPING TRISHA
AND I IN KEEPING THE COALITION
MOVING FORWARD.
PLEASE SPEND TIME WITH THEM
THEY'RE ALL WONDERFUL PEOPLE
THAT DEDICATED TIME AND ENERGY
TO THE COALITION AND WE'RE
GRATEFUL TO THEM.
>> ALSO, I WANT TO EXTEND A
SPECIAL THANK YOU TO LAUREN AND
OTHERS WHO HELPED DEVELOP THE
PROGRAM AND I'LL PUT IN
SHAMELESS PLUGS DURING THE
PRESENTATION FOR OPPORTUNITIES
TO HEAR FROM YOUR VOICES.
WE WANT TO HEAR FROM YOU.
WE WANT YOU TO JOIN US AS WE
PLAN FOR THE NEXT ONE DAY OR FOR
OTHER PROGRAMS AND ENGAGEMENT WE
WANT TO DO AT THE NIH.
PLEASE CONSIDER SHARING YOUR
VOICE IT'S REALLY IMPORTANT TO
US WHETHER YOU'RE IN A SURVEY OR
ABLE TO VOLUNTEER TO BE PART OF
THE PLANNING COMMITTEE.
IT'S A REWARDING EXPERIENCE AND
A GREAT OPPORTUNITY TO MAKE SURE
WHAT YOU'RE ORGANIZATION IS
HEARING AND HEARING FROM OTHER
ORGANIZATIONS GETS HIGHLIGHTED
TO BE SURE TO HAVE THE
CONVERSATION.
>> I PRIVILEGE OF THANKING THE
NIAMS' STAFF I'VE BEEN
OVERWHELMED WITH HOW GENEROUS
THEY ARE WITH THE TIME AND HOW
AVAILABLE THEY ARE AND THEIR
CONCERN AND INTEREST IN HAVING
THIS ORGANIZATION GROW AND
THRIVE.
PUTTING THIS COALITION MEETING
TOGETHER IS MONUMENTAL AND THE
AMOUNT OF EFFORT AND ENERGY THEY
PUT INTO IT IS JUST INDICATIVE
OF THEIR COMMITMENT TO ALL OF
YOU AND OUR ORGANIZATION.
AGAIN, THANK YOU, NIAMS'STAFF.
>> QUICKLY, TO GIVE A LITTLE BIT
OF BACKGROUND BECAUSE WE HAVE SO
MANY NEW FACES IN THE ROOM
TODAY.
I WANT TO SAY THAT'S ONE OF THE
THINGS WE'RE MOST ENERGIZED
ABOUT WHEN WE ASK WHAT GIVES YOU
HOPE FOR THE FUTURE.
ONE THING FOR US IS HOW ENGAGED
THE COMMISSION HAS BECOME.
HOW INTERESTED AND ENGAGED WE
ARE.
THIS IS A 20-YEAR-OLD COMMISSION
PUT TOGETHER BY THE
ORGANIZATIONS YOU SEE LISTED
THERE FROM A DECADE AGO WE
ORGANIZED BUT THE WHOLE GOAL OF
THIS GROUP IS TO COME TOGETHER
AND TO REALLY THINK ABOUT HOW DO
WE GET THE WORD OUT.
THIS WAS SOMETHING DR. CRISWELL
SAID IN HER TALK, HOW DO WE GET
THE WORD OUT FOR PEOPLE TO
UNDERSTAND WHAT IS IMPACTING
THOSE WE SERVE AND HOW DO WE GET
THE WORD OUT WHAT NIAMS IS DOING
TO HELP ADDRESS THAT.
>> SO WHERE ARE WE TODAY WITH
THE COALITION?
JUST A SHORT STORY, WHEN I FIRST
BEGAN MY NON-PROFIT LEADERSHIP
POSITIONS ON A NATIONAL LEVEL
MANY YEARS AGO, PROBABLY 18
YEARS AGO, I WAS AT AN INFORMAL
CONFERENCE THAT WAS HELD IN
TEXAS.
AND A GENTLEMAN THERE GOT UP TO
TALK ABOUT WHAT COALITIONS ARE
AND HOW THEY CAN BEST MOVE
FORWARD AND THEIR REAL PURPOSE.
AND SUCCINCTLY HE SAID THE
COALITIONS ARE A GROUP OF PEOPLE
WITH SIMILAR INTEREST THAT FORM
TOGETHER WHERE NO ONE PUTS THEIR
INTEREST ABOVE ANY OTHERS.
THAT WAS TRULY INSPIRING TO ME
AND THE GENTLEMAN BECAME ONE OF
MY MENTORS BECAUSE I JUST FELT
THAT FOR US IN THIS ADVOCACY
ROLE THAT WE PLAY IN NON-PROFIT
ROLES AND LEADERSHIP ROLES WE
HAVE, IT IS SO CRITICAL FOR US
TO WORK COLLABORATIVELY.
WE CANNOT ACCOMPLISH WHAT OUR
MEANS ARE AND OUR MISSION FOR
OUR ORGANIZATIONS IN A VACUUM.
WE HAVE TO WORK COLLABORATIVELY
AND HAVE TO JOIN WITH OTHERS TO
MAKE THAT HAPPEN.
THAT IS THE PURPOSE OF THIS
COALITION.
THIS -- WHAT WE REPRESENT HERE
AND I'LL USE LEE CALLAHANOVER
THERE THEY HAVE A WONDERFUL
STUDY CALLED BEMIS AND IT'S A
TRILLION DOLLAR ANNUALLY OF WHAT
THE BURDEN IS IN THE U.S. ALONE
OF A DISEASE AND WHEN YOU
COMBINE IT WITH OTHER DISORDERS
REPRESENTED HERE, WE DOMINATE
HEALTH CARE AND WE HAVE THAT
POWER AND REPRESENT THAT GROUP
OF ADVOCATES AND PATIENTS WE CAN
IMPROVE NIAMS AND GETTING MORE
FUNDING AND SUPPORTING THEIR
EFFORTS IT HELPS US AS WELL.
>> I'M GOING TO SKIP US AHEAD
QUICKLY BECAUSE I WANT TO HAVE
ENOUGH TIME TO TALK ABOUT KEY
THINGS.
THE MEMBERSHIP IS OPEN.
WE ENCOURAGE YOU TO GET THE WORD
OUT TO HAVE ADDITIONAL MEMBERS.
I'LL SKIP OVER TO THE ADVOCACY
PIECE AND SAY ONE OF THE MOST
IMPORTANT THINGS WE DO IS SHARE
OUR VOICE.
SHARE OUR STORY AND DR. CRISWELL
SAID THIS BUT I'LL KEEP
EMPHASIZING THE MORE WE TALK
ABOUT THE BURDEN THE MORE WE
MAKE IT AWARE WHAT SOME PEOPLE
LIVE WITH.
>> AND BIANNUALLY WE PUT IN TO
THE MEETING YOU'RE HERE FOR
TODAY AND WILL BE ORGANIZING OUR
CONGRESSIONAL TOUR DAY.
TO BE DETERMINED BUT PLEASE STAY
TUNED.
>> AND WHAT THINGS YOU'D LIKE TO
SEE FROM WEBINAR.
THESE ARE A FEW WE HAD AND THEY
COME FROM YOU AND WHAT THINGS
ARE IMPACTING YOU AND THE
EDUCATION AND THIS IS A GREAT
OPPORTUNITY TO ANSWER THE
QUESTION.
WE'LL ASK YOU TO THINK ABOUT
THAT AND BY THEN OF TODAY YOU
SHOULD HAVE LOTS OF IDEAS FOR
WEBINARS TO GET THOSE MOVING.
>> FOR THE COALITION ENGAGEMENT
THERE'S A LIST OF OPPORTUNITIES
THERE.
I WANT TO EMPHASIZE EVERYBODY
HERE SHOULD CONTRIBUTE TO OUR
ROUND UP AS WELL AS JOIN THE
LINKED IN PAGE BUT NOT ONLY TO
SHARE AMONG THE COALITION
MEMBERS BUT TAKE THE INFORMATION
AND SHARE IT WITH YOUR MEMBERS
AND ORGANIZATION AND RESEARCHERS
TO GET THE WORD OUT WHAT THE
COALITION IS DOING.
>> I'LL ECHO THAT BECAUSE THAT
COLLABORATIVE PIECE DR. TABAK
WAS TALKING ABOUT ONLY COMES
WHEN PEOPLE UNDERSTAND WHAT
OTHER PEOPLE ARE DOING.
SOMETIMES YOU THINK PEOPLE ONLY
CARE ABOUT CERTAIN THINGS AND
THERE'S OVERLAP IN WE'RE DOING
AND IF WE SHARE THAT OUT YOU CAN
SPARK SOMETHING IN SOMEBODY YOU
DIDN'T KNOW NEEDED THE SPARK.
WE HAVE WORKING GROUPS AND
OTHERS NOT LISTED.
WE TALKED ABOUT LEGISLATIVE
ADVOCACY WORKING GROUP AND
COMMUNICATIONS WORKING GROUP.
JOIN US.
IT'S A GREAT OPPORTUNITY FOR YOU
TO GET INVOLVED TO GET YOUR
VOICE AT THE TABLE.
>> AND PLEASE GET INVOLVED BY
JOINING THE STEERING COMMITTEE.
I'LL BE WITHOUT A CO-CHAIR SOON.
I'M HOPING SOMEBODY WILL STEP UP
TO WORK WITH ME AS DIFFICULT AS
THAT MAY SEEM AT TIMES BUT
PLEASE, WE'LL BE ACCEPTING
ANYBODY THAT'S INTERESTED,
PLEASE CONTACT US SO WE CAN HAVE
ANOTHER CO-CHAIR FOR US.
THIS IS THE WEBSITE DRAWING
ATTENTION TO IT.
A LOT HAS GONE INTO IT TO
IMPROVE IT AND MORE IS GOING TO
GO INTO IT SO IT BECOMES A
RESOURCE FOR THE COALITION.
>> AND A REMINDER TO REACH OUT
TO COLLEEN IF YOU HAVE UPDATES.
THIS SAY REALLY WELL SEARCHED
TOOL.
I WANT TO SAY THANK YOU TO THOSE
WHO HAVE LED THIS EFFORT IN
PUTTING THIS TOGETHER.
IT WAS A HERCULEAN EFFORT TO
PULL THIS TOGETHER AND WITH THAT
WE'LL PASS IT BACK TO
DR. CRISWELL FOR OUR NEXT STEP
BUT WE LOOK FORWARD TO WORKING
WITH ALL OF YOU TODAY AND
BEYOND.
>> THANK YOU SO MUCH TRISHA AND
LEE.
WHAT A FANTASTIC START TO THE
DAY.
AT THIS POINT WE'RE GOING
TRANSITION FOR A QUICK GROUP
PHOTO.
I DO WANT TO LET EVERYONE KNOW
THERE ARE BADGES THAT WERE STUCK
IN TRAFFIC THIS MORNING BUT
THEY'VE ARRIVED.
THEY'LL BE LOCATED JUST OUTSIDE
E1 AND E2 IN THE HALLWAY CLOSE
TO THE REGISTRATION TABLE.
PLEASE WAIT TO PICK UP YOUR
BADGE UNTIL AFTER THE PHOTO.
AT THIS STABLE WE'RE GOING TO
ASK OUR LOVELY NIAMS STAFF
AROUND THAT HAVE GUIDANCE HOW TO
HELP OTHERS ORGANIZE FOR THE
GROUP PHOTO TO HELP COALITION
MEMBERS, UP THE STAIRS ON THE
RIGHT-HAND SIDE WHEN YOU WALK
OUT THE DOOR HERE AND WE'RE
GOING TO GO OUTSIDE.
WE'LL HAVE OUR PHOTO OUTSIDE AND
AGAIN, NIAMS STAFF JUST HELP
EVERYONE AND OUR LOVELY
PHOTOGRAPH, LESLIE SHOULD BE UP
THERE WAITING FOR US.
I THINK THAT'S ALL FOR
ANNOUNCEMENTS.
BADGES, NIAMS STAFF, YEP, AND
PLEASE ENSURE, THERE'S A LITTLE
BIT OF A BREAK BUT TRY IT GET
BACK LEER BY 10:45.
WE HAVE OUR WONDERFUL NEXT
SPEAKER DR. GERBERDING SO THANK
YOU SO MUCH.
DR. GERGERDING IS WITH
NON-PROFIT ORGANIZATION THAT
BUILDS PUBLIC-PRIVATE
PARTNERSHIP TO SUPPORT THE
MISSION OF THE NIH.
WE HEARD SOME DISCUSSION ABOUT
THE IMPORTANCE OF THOSE
ACTIVITIES EARLIER.
SHE CO-CHAIRS THE CSIS
BIPARTISAN ALLIANCE ON GLOBAL
HEALTH SECURITY AND SHE'S A
MEMBER OF THE COMMONWEALTH FUND
COMMISSION ON A NATIONAL PUBLIC
HEALTH SYSTEM.
PREVIOUSLY, SHE SERVED AS
PRESIDENT OF MERK VACCINES AND
EXECUTIVE VICE PRESIDENT AND THE
CHIEF PATIENT OFFICER AT MERK
AND COMPANY.
FROM 2003 TO 2009 SHE SERVES ON
MANY BOARDS.
SHE'S A MEMBER OF THE NATIONAL
ACADEMY OF MEDICINE AND THE
ADJUNCT FACULTIES OF THE
UNIVERSITY OF CALIFORNIA SAN
FRANCISCO, YAY, AND CASE WESTERN
RESERVE UNIVERSITY.
FROM THEIR EARLY YEARS TO HER
CURRENT ROLE MAINTAINED KEEPING
PATIENT AS THE THE EPICENTER OF
HER WORK.
SHE VALUES PROFESSIONAL AND
PATIENT ADVOCATES SUCH AS THE
NIAMS COALITION AND AIMS TO
BRING YOUR INSIGHTS AND YOUR
VOICES INTO THE BIOMEDICAL
RESEARCH PROCESS.
SO THANK YOU SO MUCH FOR JOINING
US.
>> WHEN I SAW THE MEMBERSHIP I
WAS IMPRESSED AND AWKWARD TO
TALK ABOUT THE NIH WHICH IS IN
PART A PUBLIC-PRIVATE
PARTNERSHIP AND I FEEL HONORED
TO BE HERE AND TO HAVE TIME TO
TELL YOU ABOUT OUR WORK AND HOW
MUCH WE VALUE THE PARTNERSHIP
WITH OUR MANY PATIENT ORIENTED
ORGANIZATIONS.
ALSO I'LL SAY NIAMS IS AN
INCREDIBLE PARTNER.
WE HAVE A NUMBER OF IMPORTANT
ACTIVITIES THAT I'LL TELL YOU A
LITTLE BIT ABOUT THAT HAVE GONE
ON FOR SEVERAL YEARS AND REALLY
THE KIND OF PARTNERSHIP AND
STAKEHOLDER EVOLUTION THAT THIS
INSTITUTE OF NIH IS SO GOOD AT
IS PART OF THE REASON WHY THE
SCIENCE IN THE AREA HAS BEEN
ABLE TO ADVANCE SO FAST.
SO I'LL JUST TELL YOU A LITTLE
BIT ABOUT THE WORK THAT WE DO AT
THE FOUNDATION TO SUPPORT THE
NIH AND THEN HELP YOU KIND OF
GET INTRODUCED TO SOME BROADER
ISSUES LIKE TRUST IN SCIENCE AND
I'M SURE IT'S BEEN ON
EVERYBODY'S AGENDA IN YOUR HOME
ORGANIZATIONS AS WELL.
SO LET ME JUST START WITH OUR
THEME WHICH IS BUILDING BRIDGES
TO BREAKTHROUGH.
SO WE DO SEE OURSELVES AS TRYING
TO BE A BRIDGE BETWEEN THE
PRIVATE AND PUBLIC SECTORS AND
BUILDING BRIDGES OF SCIENCE TO
PEOPLE.
I THOUGHT IT WAS INTERESTING TO
SEE THE TAGLINE FOR THE
COALITION CONNECTING TO SCIENCE
SO IT THEMATICALLY FITS.
OUR AMBITION IS TO ACCELERATE
AND AMPLIFY THE NIH WITH BETTER
HEALTH EQUITY.
ONE IS THROUGH TEAM SCIENCE
TRYING TO BUILD THESE PLATFORMS
OF COLLABORATIVE SCIENCE AND
EXPEDITE THAT AND WE SUPPORT
SCIENTISTS AND IN THIS DAY AND
AGE WHEN EVERYONE IS WORRIED
ABOUT SUPPORT FOR POSTDOCS AND
EARLY CAREER SCIENTISTS IT'S
BECOMING AN INCREASING APPROACH
HOW WE BUILD BETTER SUPPORT FOR
SCIENTIFIC STARS.
AND LASTLY TRYING TO CONTRIBUTE
TO BUILDING THE TRUST IN SCIENCE
OR REBUILDING THE TRUST IN
SCIENCE WHICH IS THE BIGGEST
THREAT TO HUMAN HEALTH IN THE
U.S. IF NOT GLOBALLY.
I WANTED TO SHARE WITH YOU A
VIDEO THAT EXPLAINS THIS BETTER
THAN I CAN DO IT.
IF YOU BEAR WITH ME I'LL BRING
THIS UP AND YOU CAN HEAR OUR
STORY FROM OTHER SOURCES.
>> MY DAUGHTER, TAYLOR, WHEN SHE
WAS DIAGNOSED AT THE AGE OF 7
WITH CLM BATTANT DISEASE THE
GENETICIST SAYS THERE'D NOTHING
YOU CAN DO.
I KNEW I HAD A NEW GOAL IN LIFE,
IF I COULDN'T HELP MY CHILD
THERE HAD TO BE AN ANSWER FOR
FAMILIES AND CHILDREN WHO WOULD
FOLLOW MINE.
>> SCIENCE HAS THE POWER TO CURE
BUT NO SINGLE ORGANIZATION CAN
DO THAT ALONE.
AT FNIH WE BUILD BRIDGES BY
FORGE TEAMS AT THE BEST
SCIENTISTS AT NIH IN ACADEME
ANDACROSS THE BIO PHARMACEUTICAL
ECHO SYSTEM.
THROUGH THE PUBLIC-PRIVATE
PARTNERSHIP WE ACCELERATE NEW
DIAGNOSTICS, THERAPIES AND
POTENTIAL CURES.
>> FNIH HAS BEEN CHARGED BY
CONGRESS TO FILL A GAP IN THE
ECO SYSTEM OF INNOVATION.
BEFORE FNIH WAS REALLY DIFFICULT
TO ESTABLISH COLLABORATIVE
EFFORTS OF EFFORTS BETWEEN
PUBLIC AND PRIVATE SECTORS.
FNIH BRINGS ITS OWN SPECIAL
EXPERTISE TO PUTTING THESE
TOGETHER IN A WAY THAT CAN BE
PRODUCTIVE.
THE FNIH IS THE CONSUMMATE
CONVENER.
>> THAT MEANS BRINGING TOGETHER
THE BEST SCIENCE AND TECHNOLOGY,
OUTSTANDING PEOPLE WHO USED
THEIR COMBINED EXPERTISE AND
AMBITION TO ACCELERATE PROGRESS
TO PATIENTS.
THE MORE HANDS YOU HAVE ON DECK
THE MORE LIKELY YOU ARE TO
ANSWER THE VEXING PROBLEMS.
>> WE BRING TOGETHER THE
RESOURCES, WE PROVIDE THE
INFRASTRUCTURE, AND REALLY FORM
THAT BRIDGE TO THE BREAKTHROUGH.
>> AT FNIH, WE SUPPORT TEAM
SCIENCE IN SOLVING SOME OF THE
TOUGHEST BIO MEDICAL CHALLENGES.
SINCE OUR INCEPTION WE RAISED
MORE THAN A BILLION AND A HALF
DOLLARS TO SUPPORT THE MISSION
OF THE NIH AND DEPLOYED THEM FOR
PROGRAMS LIKE ACCELERATING
MEDICINES PARTNERSHIP AND THE
BIOMARKERS CONSORTIUM.
THESE PROGRAMS BRING TREATMENTS
AND MOST IMPORTANTLY HOPE TO
PEOPLE LIVING WITH CANCER,
NEUROLOGIC DISEASE AND RARE
DISEASES AND OTHER INSEEMINGLY
INCURRABLE HEALTH CONDITIONS.
>> WITH EVERY PARTNERSHIP WE
FOSTER WE'RE SEEKING TO
ACCELERATE SCIENTIFIC INNOVATION
SO WE CAN REALIZE GLOBAL HEALTH
EQUITY IN OUR LIFE TIME.
>> WE'RE WORKING WITH NIH, THE
GATES FOUNDATION AND OTHER
PARTNERS ON SOME OF THE GRAND
CHALLENGES IN GLOBAL HEALTH LIKE
MATERNAL MORTALITY, MALARIA,
TUBERCULOSIS AND HIV AIDS.
>> THE DISCOVERIES THAT HAPPEN
AT FNIH AFFECT THE HEALTH OF
EVERYONE IN THE WORLD.
>> THESE BREAKTHROUGHS WOULDN'T
HAPPEN WITHOUT WORLD CLASS
SCIENTISTS.
THAT'S WHY WE TAKE TIME TO HONER
THE CREATIVE INVESTIGATORS WHO
ARE PUSHING THE BOUNDARIES OF
POSSIBILITY WITH AWARDS LIKE THE
PRIZE IN BIOMEDICAL SCIENCE AND
TRAILBLAZER PRIZE.
>> THE FNIH HAS SUPPORTED NEARLY
25,000 SCIENTISTS OVER THE LAST
25 YEARS WITH TRAINING PROGRAMS,
FELLOWSHIPS AND AWARDS.
>> OUR PARTNERS AT NIH AND THE
AM ACADEMICS THEY ARE FROM HELP
DISCOVER MEDICINE.
FNIH BUILD THE BRIDGE FROM THE
BENCH TO THE BEDSIDE AND REDUCE
THE TIME IT TAKES TO BRING THE
PROMISING THERAPEUTICS TO
PATIENT WHO'S CAN'T WAIT.
>> TAYLOR LIVED ALMOST 13 YEARS
WITH HER DIAGNOSIS.
I WANT MOTHERS THAT FOLLOW KNOW
HAVE MORE.
THE FNIH IS RUNNING A GREAT RACE
ON BEHALF OF PEOPLE LIKE MY
DAUGHTER.
>> I KNOW OF NO OTHER
ORGANIZATION IN THE WORLD THAT
CAN DO THIS THE WAY FNIH DOES.
>> WE CAN'T DO THIS ALONE.
WE NEED YOUR SUPPORT.
SO PLEASE JOIN US IN BUILDING
BRIDGES TO BREAKTHROUGHS,
PATIENTS EVERYWHERE ARE WAITING.
>> THANK YOU.
THAT WAS AN ADVERTISEMENT IF YOU
DIDN'T GET IT.
I THINK IT DOES HELP THE STORY
THAT WE'RE A SMALL ORGANIZATION
BUT WE DO TRY TO THE CONVENER
AND AN HONOR TO BE WITH YOU
TODAY.
LET ME TALK MORE ABOUT SOME OF
THE SCIENTIFIC PROJECTS AND HOW
WE DO WORK.
IF I CAN NAME ONE THING THAT MAY
BE THE SECRET SAUCE OF THE FNIH
IS WE'RE GOOD AT PROJECT
MANAGEMENT.
AND YOU'LL SEE SOMETIMES OUR
PROGRAMS HAVE 30 OR MORE
PARTNERS AND HERDING THOSE LIONS
ISN'T EASY BUT IT'S A WAY TO
REALLY KEEP THE TRAINS RUNNING,
SET THE MILESTONES, MAKE SURE
THERE'S ONGOING PROGRESS AND TO
FACILITATE SOME OF THE PART
THINGS THAT MAKE IT DIFFICULT TO
WORK DIRECTLY WITH THE
GOVERNMENT.
I NOTICED WHEN I CAME IN THERE
WAS A COFFEE THAT SAID THE FOOD
WASN'T PAID FOR BY FEDERAL
TAXPAYER DOLLARS.
WE LIVE IN A COUNTRY WHERE JUST
IMPOSSIBLE FOR THE PRIVATE
SECTOR TO ENGAGE WITH GOVERNMENT
ORGANIZATIONS SO THAT WAS WHY
CONGRESS AUTHORIZED THE
FOUNDATION IN THE BEGINNING.
WE HAVE CRITERIA FOR THE KINDS
OF PARTNERSHIPS WE ENGAGE IN.
I THINK THE FIRST IS THE MOST
IMPORTANT.
WENT DO LIKE TO GET INVOLVED IN
THINGS THAT AREN'T REALLY
IMPORTANT AND HAVE A BIG IMPACT
ON PATIENTS.
WE'RE LOOKING FOR THINGS THAT
HAVE TO BE DONE IN A WISE CROWD
AND NOT COULD BE DONE BY A
SINGLE ORGANIZATION OR COMPANY
OR THINGS THAT ARE ALREADYING
DONE AND WE AVOID DUPLICATION
AND HAVE TO BE PARSIMONIOUS IN
THE RESOURCES WE HAVE.
WE ALSO LOOK FOR WAYS TO TRULY
LEVERAGE THE NIH CAPABILITY AND
RESOURCES.
IT'S A TRICKY SITUATION BECAUSE
IF YOU ASK THE AVERAGE AMERICAN
THEY THINK THE NIH HAS BILLIONS
AND BILLIONS WHY WOULD THEY NEED
FUNDS RAISED BY SOMEONE ELSE.
YET, ALL YOU HAVE TO DO IS LOOK
AT THE PAY LINE FOR GRANTS AND
RECOGNIZE IF WE COULD JUST HAVE
ANOTHER 10 POINTS TO GET IN THE
FUNDING RANGE THE AMOUNT OF
SCIENCE THAT GOES WASTED BECAUSE
WE CAN'T AFFORD TO PAY FOR IT SO
ANYTHING WE CAN DO TO LEVERAGE
THE OPPORTUNITY TO MOVE GOOD
SCIENCE FORWARD BY THE RESEARCH
PARTNERS IS SOMETHING THAT COMES
TO OUR POINTS OF VIEW.
HOW WE WORK IS CUSTOMIZED TO A
GREAT EXTENT.
I'M SURE BOB KNOWS THIS FROM THE
THINGS HE WORKED ON BUT WE HAVE
VARIOUS MODELS FOR HOW THE
PARTNERSHIPS COME TOGETHER.
BUT IN GENERAL, NIH RESOURCES
HAVE GRANTS AND PEER REVIEW AND
STUDY SECTION AND GRANTS GO OUT
TO THE ACADEMIES AND WE FUND
THINGS LIKE THE DATA
COORDINATING CENTER FOR OUR
PLATFORM PROGRAMS AND THEN
SOMETIMES WE SHARE RESOURCES
WITH DIAGNOSTIC COMPANIES,
ETCETERA.
IN THE MIDDLE IS A SHARED
GOVERNANCE.
IN ADDITION TO THE SECRETE SAUCE
OF PROJECT MANAGEMENT I THINK
THE SHARED MANAGEMENT IS
CRITICAL I'LL POINT OUT IN A
COUPLE PLACES.
WE TEND TO WORK IN PLATFORMS
INSTEAD OF HAVING A STABLE OF
DISPARATE PROJECTS WE HAVE A
PLATFORM APPROACH WHERE
DIFFERENT THERAPEUTIC AREAS ARE
APPROACHED IN PRETTY MUCH THE
SAME DIRECTION.
I'LL SPEND A BIT OF TIME ON SOME
OF THESE BUT THESE ARE THE MAJOR
PLATFORMS ACTIVE FOR THE COVID
PANDEMIC, ACCELERATED MEDICINES
PARTNERSHIP WHICH IS MOSTLY
ABOUT PATHWAY AND TARGET
IDENTIFICATION, ACCELERATING
CANCER THERAPIES.
THE PACT IS ONE EFFORT TO CREATE
MASTER PROTOCOLS WITH LUNG MAP
TO MOVE PATIENTS THROUGH THE
PROCESS OF GETTING THE RIGHT
CHEMOTHERAPY FOR THEIR ANSWER.
THE ADNI COLLABORATION HAS ENDED
BUT IT WAS A LONG COHORT STUDY
THAT WAS FOUNDATION TO
DISCOVERING THE BIOMARKERS THAT
HAVE ULTIMATELY LED TO BETTER
DIAGNOSTICS AND ENROLLMENT IN
CLINICAL TRIALS FOR PATIENTS
LIKELY TO PROGRESS IN THEIR
ALZHEIMER'S DISEASE.
THE GENE CONVENED AND GLOBAL
HEALTH PORTFOLIO WAS INHERITED
FROM THE GATES FOUNDATION.
THE GRAND CHALLENGE AS MANY ARE
FAMILIAR WITH THE INITIATION OF
GRANT CHALLENGES YEARS AGO AND
WHEN THEY GREW OUT OF THE GATES
FOUNDATION, MR. GATES MOVED
THOSE TO THE NIH AND THE FNIH
MANAGES THE GLOBAL PROGRAMS AND
HAVE BEEN PRODUCTIVE.
YOU MAY HAVE HEARD ABOUT THE
A-PLUS TRIAL WITH THE INSTITUTE
FOR CHILD HEALTH DEVELOPMENT IN
30,000 SOME WOMEN
INTERNATIONALLY A SINGLE DOSE
OFFER REEGEOOO --- - -ER
AND THERE'S THERE'S A BIG IMPACT
AND THE BIOMARKER CONSORTIUM
WHERE NIAMS HAS BEEN AN
INCREDIBLE PARTNER.
AND THERE'S ACCELERATED CLINICAL
TRIALS FOR THERAPEUTICS FOR THE
COVID PANDEMIC.
YOU LOOK AT THIS PANOPLY OF
PARTNERS AND IMAGINE WHAT HAD TO
HAPPEN TO DEVELOP A GREED UPON
PROTOCOLS BETWEEN FDA AND
PARTNERS AND GOVERNMENT AND
PROSECUTE BY THE NUMBERS.
800 COMPOUNDS WORE SCREENED FOR
POTENTIALLY UTILITY AND 6 OF 33
HAVE BEEN SHOWN TO HAVE CLINICAL
BENEFIT AND WE'RE NOT DONE YET.
SOME TRIALS ARE STILL ONGOING.
ONE OF MY BIG CONCERNS IS HOW DO
WE KEEP THE NETWORK OF NETWORKS
ACROSS THE 620 SITES WHO
ACTUALLY ENROLLED A TOTAL OF
MORE THAN 23,000 PATIENTS HOW DO
WE KEEP THEM WARM SO NEXT TIME
WE HAVE THE INFRASTRUCTURE READY
TO BE HEATED UP AND WE DON'T
HAVE TO START FROM SCRATCH LIKE
WE DID.
AT THE TIME THE PROTOCOL STARTED
THERE WAS NO ONE RESOURCE AT NIH
WHO KNEW ALL THE STUDY SITES AND
WHO WAS DOING WHAT.
PART OF THE EFFORT FOR THE FIRST
TIME BROUGHT TOGETHER THE
INTERNATIONAL PICTURE OF WHERE
CLINICAL RESEARCH WAS BEING
FUNDED BY NIH AND WHERE OTHER
PARTNERS COULD WEIGH IN.
THE BIOMARKERS CONSORTIUM IS
ALSO A LONG-STANDING PLATFORM
PROGRAM.
THE GOAL IS TO IDENTIFY AND
VALIDATE BIOMARKERS USED AS
SURROGATE END POINTS OR
DIAGNOSTICS TO ENRICH PATIENTS
OR PROGNOSTIC PATTERNS.
FDA IS AN IMPORTANT PARTNER IN
THIS PROCESS AND IT'S ONE OF THE
MOST FAVORED PARTS OF OUR
PORTFOLIO TO INDUSTRY PARTNERS.
IF YOU HAVE TO WAIT 10 YEARS FOR
SOMEONE TO DEVELOP A HIP
FRACTURE SO KNOW IF YOUR DRUG
WORKS TAKES A LONG TIME AND
TAKES A GAZILLION PEOPLE AND WHY
NO ONE IS DOING INNOVATIVE WORK
IN THIS SPACE OR AT LEAST
WASN'T.
IF YOU CAN VALIDATE A BIOMARKER
THAT'S A GOOD SURROGATE END
POINT FOR FRACTURE, YOU CAN GET
BACK IN THE GAME OF DRUG
DEVELOPMENT AND NEW INNOVATIONS
FOR OSTEOPOROSIS.
THAT APPROACH PLAYS OUT OVER AND
OVER AGAIN.
ONE OF OUR HOT AREAS RIGHT NOW
GOING INTO OUR NEXT AREA IS
MENTAL HEALTH.
THERE'S NO BIOMARKERS FOR MENTAL
HEALTH EXCEPT ONE FOR POSTPARTUM
DEPRESSION.
TO SAY SOMEONE'S DEPRESSED IS
LIKE SAYING SOMEONE IS HAVING
CHEST PAIN.
THERE COULD BE MANY DIAGNOSES
AND THE CONSORTIUM IS GETTING
RAMPED UP TO PARTICIPATE IN THAT
SPACE.
ACCELERATING MEDICINES
PARTNERSHIP IS OUR BIGGEST OVER
ALL PROGRAM.
THIS THE TIME LINE OF THE
VARIOUS THERAPEUTIC AREAS THAT
HAVE PARTICIPATED IN THE
COLLABORATIONS TO TRY TO
UNDERSTAND BETTER THE BIOLOGY OF
A PROBLEM OR TO UNDERSTAND THE
POTENTIAL TARGETS FOR
INTERVENTION.
ONE THING I LIKE ABOUT THE SLIDE
IS IT SHOWS GENERALLY THE
PROGRAMS ARE SORT OF DESIGN TO
OPERATE ON A FIVE TO SEVEN-YEAR
TIME HORIZON AND WHEN THE
OBJECTIVES ARE COMPLETED THE
PROJECT ENDS.
YOU NOTICE SOME GET RESTARTED
WITH PHASE 2 AND THAT'S AN
IMPORTANT INDICATOR THEY'RE
VALUABLE AND VALUABLE TO THE
PARTNERS BECAUSE THEY WOULDN'T
ANTE UP AND CONTINUE TO SUPPORT
THE COLLABORATION IF THEY DIDN'T
SEE IT CONTRIBUTING VALUE.
NEXT YEAR WE PLAN TO EVOLVE OR
LAUNCH AT LEAST FOR MORE AMPS.
THE SPI AMP SOME ARE INVOLVED
WITH SYSTEMS BIOLOGY AND THE ALS
AMP IS COMING NEXT YEAR.
I THINK WE'LL HAVE A MENTAL
HEALTH PROGRAM IN THE SPACE AS
WELL.
WE ALSO HAVE A WONDERFUL
OPPORTUNITY IN PEDIATRIC DEVICES
TO UNDERSTAND DUAL DEVICES AND
OTHER TOOLS THAT NEVER GET
STUDIED IN KIDS BECAUSE
CONDITIONS IN WHICH THEY WOULD
BE RELEVANT ARE UNCOMMON IN
CHILDREN.
THESE PROGRAMS HAVE SO FAR
EARNED $830 MILLION FROM OUTSIDE
PARTNERS AND INVOLVES MOST THE
NIH INSTITUTES.
WE HAVE A COUPLE MORE WE'RE WORK
ON TO BRING ADDITIONAL
THERAPEUTIC AREAS INTO THE
SPACE.
TO GIVE YOU A FLAVOR OF HOW
THINGS UNFOLDED, THE ALZHEIMER'S
DISEASE AMP REALLY STARTED OUT
REALLY IN A MORE TRADITIONAL
APPROACH TO LOOKING AT BRAIN
IMAGING AND FAIRLY CRUDE MARKERS
AND NOW THE 2.0 AMP IS DOWN TO
SINGLE-CELL PROFILING AND
LOOKING AT EPIGENETIC IMPACT ON
EXPRESSION OF THE DURABILITY AND
GENES THAT MAY HAVE A PREDICTIVE
OR PATHOGENIC ROLE.
THE WORK CONTINUES.
THIS IS A VERY EXCITING AREA
THAT IS KIND OF SERVING AS A
MODEL FOR HOW WE THINK ABOUT
OTHER NEURODEGENERATIVE
DISEASES.
THE TYPE 2 DIABETES PROGRAM IS
ALSO AN IMPORTANT ONE BECAUSE
IT'S SO GLOBAL.
THIS IS A KNOWLEDGE PORTAL
DESIGN INITIALLY TO LOOK FOR
GENETIC FINGERPRINTS FOR
DIABETES BUT SINCE WE'RE NOW
UNDERSTANDING THE CONVERGENCE OF
METABOLIC DISORDERS POTENTIALLY
BEING AN EXPRESSION OF UPSTREAM
SET OF FACTORS WE NEED TO
UNDERSTAND BETTER WE HAVE
EVOLVED THE TYPE 2 DIABETES
PROGRAM INTO A COMMON METABOLIC
DISEASE PROGRAM THAT'S LOOKING
SIMULTANEOUSLY AT A NUMBER OF
DISEASES.
THIS IS WHERE ONE OF THE BIGGEST
CHALLENGES THAT WE FACE COMES
INTO PLAY AND THAT IS THAT WE
DON'T HAVE DATA INTEROPERABILITY
ACROSS THE AMPS.
AS NIH GRAPPLES WITH THIS ACROSS
THE INSTITUTES WE'RE ON A SMALL
SCALE GRAPPLING WITH IT ACROSS
THE AMPS AND WE HAVE IDEAS HOW
TO CREATE A MORE FEDERATED DATA
PORTALS BECAUSE THEY'RE OPEN
ACCESS WANT PEOPLE TO BE ABLE TO
UTILIZE THEM AND INCREASING THE
UTILITY FOR HYPOTHESIS
GENERATION IF NOT ACTUAL
SCIENCE.
I'M SURE SOME ARE AWARE OF OR
PARTICIPATED IN THE AUTOIMMUNE
AND IMMUNE-MEDIATED DISEASE AMP.
THIS IS EVOLVING TO THE NEXT
GENERATION AND AFTER THAT.
I WON'T GO IN THE DETAILS BUT
BUT I THINK IT'S ANOTHER EXAMPLE
OF CONVERGENT THINKING.
WHAT I DID WANT TO SPEND A
COUPLE MINUTES ON IS THE BESPOKE
GENE THERAPY CONSORTIUM WHICH IS
ALSO AN AMP.
THE GENE THERAPY CONSORTIUM IS A
LITTLE BIT UNIQUE IN OUR
PORTFOLIO.
OBVIOUSLY THE GOAL IS TO GO
AFTER RARE DISEASES WITH A
GENETIC BASIS.
THE INITIAL PROGRAM IS FOCUSSING
ON AAV VECTORED GENE THERAPIES.
IT'S ACTUALLY AN AMP THAT IS NOT
SO MUCH ORIENTED TOWARDS SOLVING
THE UNIVERSE OF GENETIC DISEASES
BUT AN AMP INTERESTED IN TRYING
TO FIGURE OUT HOW TO CREATE A
REGULATORY FRAMEWORK FOR
MANUFACTURING OF THESE BIOLOGIC
S SO EVERY COMPANY WITH A GOOD
IDEA FOR A GENE THERAPY DOESN'T
HAVE TO START FROM SCRATCH.
EVERYBODY'S MIXING THIS UP IN
THEIR BATHTUB AND IT WOULD BE
NICE IF THERE WAS A MORE
STANDARDIZED APPROACH TO
ACCELERATE THE AVAILABILITY AND
TAKE THAT DEGREE OF UNCERTAINTY
OUT OF IT FOR THE MANUFACTURERS.
LIKEWISE WE NEED MORE REGULATORY
CERTAINTY HOW TO PROSECUTE
ADVANCED STAGE CLINICAL TRIALS
WHEN YOU CAN'T DO A CLINICAL
STUDIES BECAUSE THERE'S NOT
ENOUGH PATIENTS TO THAT STUDY
DESIGN MAKE SENSE.
THE FIRST AID CONDITIONS MAY BE
OF INTEREST TO SOME OF YOU ON
THE RIGHT.
THEY WERE ANNOUNCED THIS YEAR.
THE MANUFACTURING PROCESS ARE IN
PLAY AND WE'RE GETTING THE
CONTRACTS ORGANIZED WITH
INDUSTRY PARTNERS.
I LOVE THE QUOTE BECAUSE MY
COUSIN'S KID HAS MUSCULAR
DISTROPHY AND I KNOW ABOUT BAKE
SALES AND WALKS AND HOW HARD IT
IS TO RAISE MONEY FOR DISEASES
TO KNOW THERE'S BEEN TRUE
INVESTMENT AND WE CAN MAKE IT
EASIER FOR THE BIO SCIENCE
PARTNERS TO BE WILLING TO PUT
FORWARD THE RESOURCE MEANS
EVERYTHING.
THE TEAM DECIDED WE'D MOVE INTO
NON-VECTOR GENE THERAPIES AND
WORKING WITH INDUSTRY PARTNERS
KNOWN AS HEVE.
THE HEADS OF R AND D FROM THE
MAJOR PHARMACEUTICAL COMPANIES.
WE BROUGHT THE NOTION OF AN AMP
THAT LOOKED BEYOND AAV AND
THERE'S OTHER VIRAL AND
NON-VIRAL VECTORS AND CRISPR
TECHNOLOGY WE CAME TO CONCLUDE
IT WAS TIME TO MOVE PAST THE ONE
POINT APPROACH.
THAT WOULD BE A NEXT CHAPTER OF
THIS AS WELL AND THERE'S NON-NIH
AND GOVERNMENT PARTNERS AND CIM
IS THE CALIFORNIA INSTITUTE FOR
REGENERATIVE MEDICINE A
QUASI-GOVERNMENTAL ORGANIZATION
AND YOU SEE LITTLE
ORGANIZATIONS, BIG COMPANIES,
PATIENT ORGANIZATIONS, A WHOLE
SET OF STAKEHOLDERS WORKING
COLLABORATIVELY ON THE PROGRAMS.
AND BILL AND MELINDA GATES
LOOKED AT IN VIVO AND EX VIVO IN
DEVELOPING COUNTRIES WHERE MOST
SICKLE CELL DISEASE AND TO
ADVANCE TRUST IN THE COMMUNITY
AND THE SCIENTIFIC CAPABILITY TO
WORK ON A PROBLEM LIKE THIS IS A
LONG RUNWAY BUT WORTH DOING AND
SUCH A DISEASE OF YOUNG KIDS IN
THE AREAS IT SEEMS LIKE A
PROGRAM WORTH PURSUING.
THE BIOMARKERS PROGRAMS ARE AN
AMP WE'RE LAUNCHING NEXT YEAR
ARE LISTED HERE AND ONE THING
ABOUT THE ALS PROGRAM BECAUSE
CONGRESS PUT I THINK $100
MILLION IN THE CONGRESSIONAL
BUDGET FOR ALS.
THAT HAS MADE THIS A FEASIBLE
PLACE FOR ENGAGEMENT AND THE
INDUSTRY PARTNERS HAVE REALLY
STEPPED UP.
WE'RE STARTING TO UNDERSTAND THE
BIOLOGY BUT IT'S ALSO A
WONDERFUL MIXTURE OF SCIENCE AND
ADVOCACY INCLUDING DR. DOTOROV
IS A FORMER DEPUTY MAYOR OF NEW
YORK CITY BUT ALSO A PERSON
WHOSE FATHER AND UNCLE DIED OF
ALS ON A GENETIC BASIS AND NOW
HE HAS ALS NOT GENETIC AT LEAST
NOT THE SAME GENE THEY DO.
IT'S MYSTERIOUS.
HIS DISEASE IS PROGRESSES BUT
STILL BUZZES AROUND ON HIS VESPA
MOTORCYCLE AND GENE TO BE THE
ENERGIZER AND WE ENGAGE DIRECTLY
AND THE OTHER THING ABOUT THE
AMP ALS I'M EXCITE ABOUT IS WHEN
I WAS LEARNING ABOUT FNIH AND
NIH AND SPEAKING TO EXTERNAL
STAKEHOLDERS THE ONE THING THAT
DISAPPOINTED MOST IS BECAUSE
WE'RE FLOW.
FNIH DOESN'T START WORK UNTIL WE
FULLY FUNDED I.
WE RAISE ALL THE MONEY BECAUSE
WE COULDN'T AFFORD TO BE CAUGHT
HATHAWAY WITHOUT BEING ABLE TO
COMPLETE THE JOB.
WE'RE WORKING ON WAYS TO SPEED
THIS UP.
AND SOME HAS TO DO WITH SPEEDING
UP THE CONCEPT AND DEVELOPMENT
PHASE DOING IT ALL AT ONCE AND
WE'LL HAVE A CALL THIS MONTH AND
THEN ENERGY CALL MONTH AFTER
MONTH AND LET'S SIT DOWN AND GO
WITH IT.
WE'RE LAUNCHING A SIGNIFICANT
FUND RAISING PROGRAM AND THAT
VIDEO IS A TOOL WE'LL BE USING
BUT THE IDEA IS IF WE HAD A POOL
OF FLEXIBLE RESOURCES WE COULD
JUMP START THESE THINGS.
IF FNIH HAS A CONTINUING
RESOLUTION AND CAN'T GET A NEW
PROGRAM STARTED BECAUSE THEY
CAN'T FUND IT THROUGH THE
CONGRESSIONAL AUTHORIZATION, WE
CAN START IT IF WE HAVE HIGH
CONFIDENCE EVENTUALLY IT WILL BE
FUNDED.
WE'RE WORKING ON HOW WE CAN
IMPROVE OUR OWN PROCESSES AND
HOW WE CAN ADDITIONAL RESOURCES
SO WE CAN MOVE FOMORE QUICKLY
BECAUSE PATIENTS ARE WAITING.
AND ONE OF THE THINGS WE'RE
DOING WHICH IS REQUIRING A LOT
FROM THE FDA IS WORKING ON
DIGITAL BIOMARKERS.
AND HOW TO USE THEM RELIABLELY
IN AN FDA APPROVED MANNER FOR
CLINICAL TRIALS.
EVERYONE'S USING DIGITAL DEVICES
BUT THERE'S NO STANDARDIZATION.
IF YOU'RE A CLINICIAN YOUR
PATIENTS ARE CONSTANTLY COMING
IN WITH THEIR SLEEP RING
PRINTOUT OR HEART RATE
INFORMATION, ETCETERA.
YOU CAN'T EXPECT THIS TO BE
PARTICULARLY HELPFUL FOR FDA
DRUG APPROVAL OR DEVICE APREFL
FOR THAT MATTER WITHOUT SOME
DEGREE OF STANDARDIZATION.
WE'RE WORKING CLOSELY WITH FDA
TO FIGURE OUT HOW WE MIGHT BE
ABLE TO CREATE INTEROPERABLE
STANDARDS AND WHEN WE WERE
IMAGING FOR ALZHEIMER'S SIEMENS
AND IBM HAS DEVICES AND THAT
PROVED TO BE VALUABLE TO THE
INDUSTRY PARTNERS AND TO THE
FDA.
I SUSPECT SOME SIMILAR MECHANISM
AND WE HAD A CONVENING ON
BIOMARKERS AND I THINK IT WOULD
BE RELEVANT TO THE DISEASE AREAS
NIAMS IS FOCUSSED ON.
I WANT TO SAY SOMETHING ABOUT
PATIENT CENTRICITY.
NIH IS PATIENT CENTRIC AND I WAS
AT MERCK AND WE LOOKED AT
PATIENT OUTCOMES BUT WE CREATED
A POSITION FOR AN EMPLOYEE ONE
OF OUR PROJECT OFFICERS BUT ALSO
IS NOW OUR CHIEF PATIENT
ORGANIZER.
OVER THE LAST YEAR WE'VE DONE
THINGS TO TRY TO BRING THE
VOICES OF PATIENTS IN A MORE
DISTRICT WAY INCLUDING LAUNCHING
A PATIENT ENGAGEMENT COUNCIL
WHICH INCLUDES A NUMBER OF
PATIENT ADVOCATES.
MORE IMPORTANTLY, INCORPORATING
AS PROJECTS ARE DESIGN THE INPUT
OF THE PATIENT ORGANIZATIONS AND
INDIVIDUALS WHO ARE MOST
INTERESTED IN HOW THE PROGRAMS
PROGRESS.
WE DO NOT -- I DO NOT APPROVE OF
A NEW PROJECT UNLESS THE PLAN
INCLUDES IN A MEANINGFUL WAY NOT
JUST CHECK A BOX, WE TALK TO A
PATIENT BUT MEANINGFUL INPUT AND
WE'RE LEARNING AND THAT'S ONE OF
THE THINGS THE PATIENT
ENGAGEMENT COUNCIL IS HELPING US
WITH WE ALSO HAVE A NUMBER OF
PATIENT AMBASSADORS AND WE HAVE
PATIENT ARE MONTHLY ALL HANDS
MEETING TO COME IN AND SHARE
WITH US THEIR LIVED EXPERIENCE
WITH THE DISEASES.
WE HAD SOMEONE COME SPEAK ABOUT
THEIR EXPERIENCE WITH ALS.
AND SOME 25,000 PEOPLE
PARTICIPATED IN VARIOUS TRAINING
AND MENTORSHIP AND FELLOWSHIP
PROGRAMS BUT OUR MOST RELEVANT
VISUAL CONTRIBUTION ARE OUR
TRAILBLAZERS.
THE PEOPLE WHO ARE SINGLED OUT
FOR BEING RISING STARS OUR
ANNUAL CEREMONY TO HONOR THE
INDIVIDUALS IS COMING FORWARD IN
OCTOBER.
FOR SOME REASON SEVERAL OF THE
CANDIDATES AND TWO OR THREE OF
THE PEOPLE WHO ARE GOING TO BE
HONORED ARE INVOLVED IN
MITOCHONDRIAL RESEARCH SO GUESS
WHAT WE MIGHT BE WORKING ON NEXT
YEAR?
THE TIME HAS COME.
I'VE TALKED ABOUT TEAM SCIENCE
AND SUPPORT FOR SCIENCE AND END
WITH THE NOTION THAT IS TO TELL
YOU ABOUT EARNING TRUST IN
SCIENCE AND THE COALITION FOR
TRUST IN HEALTH AND SCIENCE
THAT'S BEEN ESTABLISHED.
THERE'S NOW MORE THAN 80
ORGANIZATIONS THAT HAVE SIGNED
ON TO THE COALITION INCLUDING
SOME THAT ARE INCLUDED IN THE
COALITION HERE.
THE PURPOSE OF THIS IS TO
RECOGNIZE EVERYONE'S TRYING TO
DO SOMETHING ABOUT
MISINFORMATION AND
DISINFORMATION AND TRUST BUT
WE'RE NOT ADDING UP TO HAVING
MUCH IMPACT BECAUSE EVERYBODY'S
DOING THEIR THING AND THEIR
SPACE AND THE VECTORS ARE NOT
POINTING IN THE SAME DIRECTION.
SO THIS ORGANIZATION HAS COME
TOGETHER TO JOIN YOU HAVE TO
AGREE TO DO THREE THINGS.
ONE, AGREE TO CONTRIBUTE
INFORMATION ABOUT WHAT YOU ARE
DOING AND WHETHER OR NOT IT'S
WORKING TO A LEARNING COMPENDIUM
SO THAT HOPEFULLY AT A LOCAL
LEVEL PEOPLE CAN CONNECT UP WITH
OTHERS WORKING ON THE SAME SORT
OF THINGS.
AND AGREE WHEN YOU HEAR
MISINFORMATION YOU'LL TAKE STEPS
TO CORRECT IT PREFERABLY ON
SOCIAL MEDIA BECAUSE THAT'S
WHERE MOST THE DISINFORMATION IS
CIRCULATING AND PUSHBACK ON
DANGEROUS DISINFORMATION.
THAT'S ONE PLACE WE'RE TRYING TO
HELP TO GIVE PEOPLE INFORMATION
AND WHEN YOU ADD UP THE PEOPLE
IN THE ORGANIZATIONS INCLUDING
THE TRIPLE AAAS WE'RE TOUCHING
HUNDREDS OF THOUSANDS OF MEMBERS
IN COMMUNITY ORGANIZATIONS.
IF WE MOBILIZE OUR OWN CHANNELS
ON SOCIAL WE'D BE A POWERFUL
SOURCE OF INFORMATION AND THAT'S
THE VISION HERE.
I WANTED TO MAKE YOU AWARE OF IT
AND SOME OF YOU MAY HAVE A
PARTICULAR INTEREST IN THIS
SPACE.
THE HEALTH RESEARCH ALLIANCE IS
A MEMBER AS ARE SEVERAL OTHER
OHS.
I'LL END THERE AND SAY WE ARE
AMBITIOUS AND EXCITED I LOVE
GOING TO WORK EVERY DAY IT'S THE
MOST FUN I'VE HAD AT A JOB EVER
AND GOOD TO BE CLOSE TO THE
SCIENCE AGAIN AND WE HAVE WORK
TO DO AND HOPE YOU'LL BE PART OF
THE BRIDGE.
THANK YOU VERY MUCH.
QUESTIONS OR COMMENTS?
>> MY QUESTION IS RELATED TO
ENGAGEMENT OF UNIVERSITIES
DIRECTLY.
IT SEEMS LIKE YOU'RE ENGAGING
ASPECTS OF THE HEALTH SCIENCE
CONTINUUM AND THERE'S MEDICAL
CENTER AND UNIVERSITIES
RECEIVING RESOURCES FROM NIH.
DO YOU DO ANYTHING TO ENGAGE
WITH THEM OR WHAT'S THAT LOOK
LIKE IN TERMS OF COLLABORATION?
>> I VISITED BAYLOR MEDICAL
CENTER AND WE MET WITH THE CEO
AND THAT'S ACTUALLY WHERE I
FIRST REALLY BECAME CONVINCE THE
NUMBER ONE ISSUE IS THE RESEARCH
IS POSTDOCS.
AND THE NIH ADVISORY COMMITTEE
HAS SPECIAL WORKING GROUP ON
POSTDOC SUPPORT AND I HAD A
CHANCE TO INTERACT WITH THEM AND
MAY INCREASE TRAINING AND
ACADEMIA AND TRAINING AND
INDUSTRY COULD FACILITATE EYE
SOLUTION THAT COULD BE WIN-WIN
AND NOT JUST ALLOW INDUSTRY TO
HARVEST THE BEST POSTDOCS.
IN THE CONVERSATIONS AND THERE'S
A HUGE RESEARCH LAB SO THEY'RE
INTERESTED IN HOW THE
PARTNERSHIP MODELS WORK AND THEY
ASKED THE EXACT QUESTION YOU
DID.
WE DEVELOPED A LEGAL OPINION
WHICH SAYS WE'RE NOT PROHIBITED
FROM DOING IT AND NOT
SPECIFICALLY AUTHORIZED.
MAKE SURE WE DON'T ARE UP AFOUL.
I THINK WE CAN MOVE IN THAT
DIRECTION AS WELL.
THAT MOVES FOR ACADEMIA BECAUSE
THEY'RE TRYING TO BUILD
PUBLIC-PRIVATE PARTNERSHIPS.
AS LONG AS WE DO IT WITH THE
FULL INCLUSION OF NIH AND
CONVERSATION AND FULL
TRANSPARENCY IT COULD BE
HELPFUL.
THE SMALLER COMPANIES ARE
INTERESTED IN THIS.
IS ANYONE HERE FROM IC?
THE NIAID EFFORT TO DO THE
COUNTER MEASURE DEVELOPMENT THE
STATE HAS PUT MONEY INTO THAT
BUT THEY'RE RAISING MON Y FROM
THE PRIVATE SECTOR TO DO THE
TRANSLATIONAL PIECE AND THE
MODEL THAT LOOKS SOMETHING LIKE
THIS.
I CO-CHAIRED A COMMISSION AND
IT'S A MODEL WE MAY BE ABLE TO
EXPAND.
>> HOW DO SPIRs AND STPRs GET
[INAUDIBLE]
>> I'M NOT AN EXPERT ON THOSE
GRANTS BUT I READ THE ONE THAT
JUST CAME OUT AND THEY GO TO A
SINGLE ENTITY AND WE WOUL NOT
REALLY WANT TO HAVE A SINGLE --
I'M SORRY, I NEED TO TURN THIS
ON.
OUR INDUSTRY MODEL IS
MULTIPLE INDUSTRIES.
WE LIKE TO HAVE MULTIPLE
PARTNERS AND ALSO IP IS NOT OUR
GOAL.
THESE ARE PRE-COMPETITIVE
COLLABORATIONS AND THAT MAKES IT
DIFFERENT.
DO YOU WANT TO SAY ANYTHING
ABOUT THE SBIR MODEL BECAUSE I
DON'T KNOW THE OTHER
DISTINCTIONS.
WE'RE AFFORDED A CERTAIN
PROPORTION OF OUR BUDGET THAT
GOES WITH SMALL BUSINESS ARE
INTERESTED IN DEVELOPING A
DEVICE OR APPROACH AND WE REVIEW
IT AS A GREAT COMPLIMENT TO SOME
OF THE OTHER WORK WE DO BUT I
AGREE IT'S A DIFFERENT MODEL
THAN THE FNIH MODEL IN TERMS OF
THE STAGE OF THE PROCESS AND THE
COLLABORATIVE MULTI-PARTNER
NATURE.
IF ANY OF MY COLLEAGUES WANT IT
ADD TO THAT, PLEASE FEEL FREE.
>>
[OFF-MIC]
-- SO THE QUESTION I WAS ASKING
IS OPPORTUNITIES FOR PROJECTS
WITHIN THE FNIH TO BE PASSED TO
OTHER PARTNERING S WITH THE FNIH
AND THE AMP AIM PROGRAM IS
CREATING A TREMENDOUS AMOUNT OF
INFORMATION ON TARGETS FOR
POTENTIAL BIOMARKERS THAT I
WOULD LOVE TO SEE WORKING INTO
THE BIOMARKERS CONSORTIUM.
DOES SUCH A STRUCTURAL MECHANISM
EXIST WITHIN THAT AND ARE THERE
OPPORTUNITIES FOR GROUPS LIKE
OURS TO ADVOCATE WHEN THOSE
OPPORTUNITIES MIGHT PEAK
INTEREST?
>> I UNDERSTAND YOUR QUESTION
AND THE ANSWER IS YES BUT NOT
SYSTEMICALLY.
IT'S NOT LIKE WE HAVE A PLANNED
ARCHITECTURE TO DO IT.
ONE EXAMPLE IS ALZHEIMER'S
DISEASE WHERE IT START AS A
LONGITUDINAL COHORT AND
BIOMARKERS WERE DISCOVERED AND
THEY WENT INTO THE BIOMARKERS
CONSORTIUM FOR THAT DISEASE AND
WE HAVE OBVIOUSLY AN ALZHEIMER'S
AMP AS WELL AND THIS IS THE
CHALLENGE I MENTIONED THE
INTEROPERABILITY CHALLENGE AND
AS WE THINK MORE SYSTEMS BILLING
AND LESS ABOUT A SPECIFIC
THERAPEUTIC AREA WE'RE GOING TO
NEED TO FIGURE THIS OUT.
BECAUSE IN TIMES THE PARTNERS
AND PEOPLE ARE THE SAME
PARTICULARLY ON THE INDUSTRY
SIDE YOU CAN IMAGINE THERE'S
ALREADY AN ELEMENT OF
INTEGRATION BUT THE DATA SYSTEMS
WERE NOT DESIGN UP FRONT TO MAKE
THAT EEFBL.
-- FEASIBLE.
WE'RE THINKING SOME OF THE WORK
BEING STARTED AS PHASE 1 STUDIES
OF EXISTING DRUGS THAT ARE
APPROVED FOR SAY DEPRESSION BUT
FOR SUBTYPES OF PEOPLE THAT
RESPOND IF WE COULD DESIGN THE
DATA TO ACCOMMODATE THAT WITH
THE IDEA IN MIND THAT WE'RE
GOING TO BE MOVING THIS FORWARD
INTO A BC PROJECT OR AMP WE
WOULD DESIGN THE DATA COLLECTION
AS CLOSE TO WHAT WE THINK WOULD
BE THE FUTURE OF THAT NEED GOING
FORWARD.
AND IT'S PART OF TO BE CANDID IS
THAT INSTITUTE HAVE THEIR OWN
APPROACH HOW THEY MANAGE DATA.
WE DID A CROSS-WALK OF ALL THE
PROGRAMS SOME ARE AT SAGE, SOME
MANAGED AT THE NIH AND SOME AT
ACADEMIC DATA CENTER AND COMING
UP WITH A COMMON CORE DATA SET
FOR STANDARDS THAT DON'T HAVE TO
DO WITH THE OMICS SITUATION IT'S
A BIG LIFT.
I THINK NCI AND MONICA ARE VERY
INTERESTED IN THIS SO IT MAY BE
SNAG WILL GEZ -- GET EASIER IN
THE FUTURE.
I THINK THE AMPS COULD BE A TEST
BED OR THE NEW AMPS COMING WE
MAY BE ABLE TO FACILITATE THAT
BUT IF YOU HAVE IDEAS TALK TO
BECAUSE I'M TALKING ABOUT USE
CASE.
>> I LOVE THE LINE ABOUT HERDING
LIONS.
>> I THINK MY TIME IS -- SHE'S
HOLDING ME THE ONE MINUTE TO END
SIGN.
I'LL AGAIN THANK YOU FOR THE
WORK YOU DO AND FOR THOSE
CLOSEST TO THE PATIENTS THANK
YOU FOR BEING ADVOCATES.
I WAS GLAD TO SEE THAT WAS AN
IMPORTANT PART OF THE
COALITION'S WORK BECAUSE WE
CAN'T DO THAT AND WE'RE GLAD
THERE'S A UNIVERSE OF PEOPLE WHO
ARE BECAUSE NIAMS NEEDS YOUR
SUPPORT.
THANK YOU.
>> THANK YOU DR. GERBERDING AND
FOR TAKING THE TIME TO BE WITH
US TODAY.
I'M PLEASED TO KICK OFF THE NEXT
SESSION OUR LAST SESSION OF THE
MORNING BY INTRODUCING DR. GAIL
LESTER DIRECTOR OF THE DIVISION
OF EXTRAMURAL RESEARCH AT NIAMS
WHO WILL KICK OFF THE NEXT
SESSION TODAY.
IN ADDITION TO HER PROGRAMMATIC
MANAGEMENT OF GRANT, SHE SERVES
AS THE PROJECT OFFICER FOR THE
PUBLIC-PRIVATE PARTNERSHIP
OSTEOARTHRITIS INITIATIVE AND AS
THE PROJECT DIRECTOR FOR THE
FNIH BIOMARKER CONSORTIUM BONE
QUALITY PROJECT.
PRIOR TO HER ARRIVAL IN NIAMS IN
2001 SHE WAS A PROFESSOR IN THE
DEPARTMENT OF ORTHOPEDICS AT THE
UNIVERSITY OF NORTH CAROLINA AT
CHAPEL HILL AND AUTHORED MORE
THAN 60 PEER-REVIEWED
PUBLICATION AND SEVERAL BOOK
CHAPTERS FROM THE SEGMENT OF HER
CAREER.
OVER TO YOU.
>> HELLO, I'M DELIGHTED TO GIVE
YOU THE INTRODUCTION TO NIAMS
EXTRAMURAL PROGRAMS AND THOSE
HERE TO TALK ABOUT THE SCIENCE
WE SUPPORT AT NIAMS.
MY TALK IS GOING TO BE PRETTY
SHORT.
I ONLY HAVE EIGHT SLIDES AND
SEVERAL ARE NOT SPEAKABLE TO
LIKE THIS ONE BUT I'LL TRY TO
MOVE THROUGH RAPIDLY SO WE HAVE
MORE TIME TO BE TOGETHER IN THE
GROUPS THAT WE'RE GOING TO GO
INTO AS SOON AS I FINISH
PRESENTING TO YOU.
YOU ALREADY HAD AN INTRODUCTION
FROM DR. CRISWELL ABOUT THE
MAKEUP OF NIAMS FOR THOSE WHO
MAY BE HERE FOR THE FIRST TIME,
IT'S IMPORTANT FOR YOU TO
UNDERSTAND OUR MISSION WHICH IS
OUTLINED IN THE THREE BULLETS
AND TO UNDERSTAND THAT
APPROXIMATELY 83% OF THE FUNDING
TO THE INSTITUTE GOES TO THE
EXTRAMURAL RESEARCH COMMUNITY.
THAT'S WHERE WE ARE REALLY
CHARGE WITH ENSURING THE WORK
SUPPORTED IS OF HIGH QUALITY AND
CONTRIBUTING TO OUR
UNDERSTANDING OF DISEASE AND
PATIENT CARE.
SO TO GIVE YOU AN IDEA OF WHAT
THE MAKEUP OF THE INSTITUTE IS,
I AM THE DIRECTOR OF THE IS
THERE A POINTER?
I AM THE DIRECTOR OF THE
EXTRAMURAL RESEARCH PROGRAM AND
THE AND THE ENTIRE EXTRAMURAL
RESEARCH IS MADE UP OF TWO
DIVISIONS.
ONE IS THE EXTRAMURAL RESEARCH
GROUP I LEAD AND THE OTHER ONE
IS THE EXTRAMURAL ACTIVITIES.
THESE ARE REALLY BOTH CRITICAL
COMPONENTS TO ENSURING NOT ONLY
THE SCIENTIFIC QUALITY OF THE
RESEARCH IS CARRIED OUT BUT IN
THE EXTRAMURAL ACTIVITIES THE
MANAGEMENT OF CLINICAL TRIALS,
THE MANAGEMENT OF THE GRANT
FINANCIAL ASPECTS AND THE
SCIENTIFIC REVIEW BRANCH ARE
REALLY CRITICAL COMPONENTS TO
ENSURE WHAT'S HAPPENING ON THE
OTHER SIDE IS CARRIED OUT
APPROPRIATELY.
WHAT THE RESPONSIBILITY OF
PROGRAM?
I OVERSEE THE PROGRAM S WHO WORK
IN PARTICULAR SCIENTIFIC AREAS.
THESE SCIENTIFIC AREAS ARE
CLINICALLY OR BASIC SCIENCE
ORIENTED AND SOME PROBABLY A
LITTLE BIT OF BOTH AND ALL OF
THEM SEEM TO HAVE A LITTLE BIT
OF A TRANSLATIONAL COMPONENT.
EVEN IF A PROGRAM DIRECTOR IS
MANAGING A BASIC SCIENCE
COMPONENT, THERE'S GOING TO BE
THOUGHT ABOUT HOW THIS COULD
IMPACT DISEASE THE DISCOVERIES
IN THE BASIS OF DISEASE AND
HEALTH.
THE RESPONSIBILITY OF THE
PROGRAM DIRECTORS INVOLVE
SEVERAL ASPECTS.
THEY PROVIDE TECHNICAL
ASSISTANCE TO THE APPLICANTS.
THEY OVERSEE WHATEVER COMES TO
US IS APPROPRIATELY ASSIGNED TO
THE NIAMS.
WE HAVE 27 INSTITUTES AND
CENTERS AT NIH AND THERE'S A LOT
OF OVERLAP BETWEEN THESE.
WORK WITH THE OTHER PROGRAM
DIRECTORS AT OTHER INSTITUTES
AND CENTERS AND ENSURE OUR
ASSIGNMENTS ARE APPROPRIATELY
MADE AND THEY ALSO OBSERVE THE
SCIENTIFIC REVIEW PROCESS THAT
EVERY APPLICATION COMES IN IS
ACCEPTED AND UNDER GOES A
RIGOROUS SCIENTIFIC PEER REVIEW
PROCESS.
THEY MANAGE ANY QUESTIONS THAT
COME UP FROM THE APPLICANTS AND
ALSO PREPARE FUNDING
RECOMMENDATIONS BASED ON THE
QUALITY OF THE APPLICATIONS FROM
THE PEER REVIEW PROCESS.
ONCE FUNDING IS DONE AND THE
APPLICANTS RECEIVE FUNDS TO
CARRY OUT THE RESEARCH THEY
PROPOSED, THE PROGRAM DIRECTORS'
JOBS INCLUDE MANAGING ANY ISSUES
THE APPLICANTS RUN INTO WITH
REGARDS TO MAKING CHANGES IN THE
SCIENCE THAT MAYBE GO FROM ONE
MODEL TO ANOTHER BECAUSE THE
FIRST ONE DIDN'T WORK OR AND
THEY MONITOR THE PROGRESS PER
YEAR BECAUSE OUR FUNDING IN MOST
CASES IS YEARLY ONE OR TWO, FIVE
YEARS.
THOSE TYPES OF INTERVALS BUT
EVERY YEAR YOUR PROGRESS AS AN
APPLICANT RECEIVING FUNDS IS
REVIEWED BY THE PROGRAM
DIRECTORS.
POST FUNDING THEY WOULD BE
LOOKING AT THIS PROGRESS BUT
THEY ALSO GO TO MEETINGS AND
LOOK FOR PRESENTATIONS FROM THE
APPLICANTS AND EVALUATE HOW THE
SCIENCE THAT WE HAVE BEEN
FUNDING AS AN INSTITUTE FITS
INTO THE BIGGER PICTURE OF
WHAT'S BEING DONE IN THE OVER
ALL BOTH U.S. AND INTERNATIONAL
SCIENTIFIC AREAS.
HERE'S OTHER THINGS PROGRAM
DIRECTORS DO AND WHY IT'S
IMPORTANT FOR TO YOU MEET WITH
THEM TODAY.
THEY'RE THE PUBLIC FACE OF THE
GOVERNMENT.
THEY ATTEND SCIENTIFIC MEETINGS
AS I JUST MENTIONED AND ALSO
INTERACT WITH PUBLIC AND
VOLUNTARY ORGANIZATIONS SUCH AS
THE COALITION AND ARE VERY
IMPORTANT SOURCES OF INFORMATION
FOR PEOPLE LIKE YOU IF YOU'RE
CURIOUS ABOUT WHAT THE STATE OF
SCIENCE IS.
THEY CAN HELP YOU UNDERSTAND
WHERE THE NEEDS AND
OPPORTUNITIES ARE.
.
THEY FOLLOW THE BIOMEDICAL
DEVELOPMENTS AND ADVANCES AND
PROVIDE ADVANCES BASED ON WHAT
RESEARCH IS PUBLISHED ON A
TRI-YEARLY BASIS SO THE
INSTITUTE SO WE'RE UP TO DATE
WITH WHAT THE ACTIVITY IN EACH
INDIVIDUAL AREA OF RESEARCH.
THEY INITIATE SPECIFIC PROJECTS
AND INITIATING.
THEY DO THAT AND THESE
INITIATIVES ARE CONSIDERED BY
THE ENTIRE LEADERSHIP AND
PROGRAM DIRECTORS STAFF OF THE
INSTITUTE AND APPROVED FOR SENT
BACK FOR MORE WORK BASED ON THE
NEED AND THE CAPABILITIES TO
BRING IN SCIENCE AND IN THAT
PARTICULAR AREA.
THEY ALSO SERVE ON TRANS-NIH
COMMITTEES.
ONE OF THE THINGS WE HEARD IS
THERE'S LOTS OF INSTITUTES AND
CENTERS AND THEY'RE ALL DOING
REALLY EXCITING RESEARCH.
IN SOME CASES LIKE DR. CRISWELL
MENTIONED THE HE'LL INITIATIVE
OR THE AMP, THESE ARE MULTI-I.C.
INITIATIVES.
WORKING WITH YOUR COLLEAGUES AS
AN NIH EMPLOYEE IS VERY
IMPORTANT AND HELPFUL TO THE
INSTITUTE.
WE WORK WITH NEW INVESTIGATORS
AND WITH BRINGING IN MINORITY
INVESTIGATORS AND HELPING THEM
MENTORING THEM THROUGH PROVIDING
RESOURCES THAT WE'RE CAPABLE OF
DOING NOT FINANCIAL BUT OTHER
POINTING THEM IN THE PROPER
DIRECTIONS.
PART OF THIS IS ACCOMPLISHED
THROUGH OUR DIVERSITY SCHOLARS
PROGRAM FOR DIVERSITY
SUPPLEMENTS TO NIH GRANTS AND
OTHER THROUGH THE K FORUM WHERE
WE HAVE INVESTIGATORS WHO ARE
AWARDED CAREER DEVELOPMENT
AWARDS AND ARE BROUGHT TOGETHER
IN THE SECOND, THIRD OR FOURTH
YEAR OF THEIR AWARD AND MEET
WITH OTHERS AND SPEND TWO DAYS
WITH US AT THE INSTITUTE WITH
DR. CRISWELL AND DR. CARTER AND
ALL THE STAFF TO WORK WITH THEM
AND HELP THEM UNDERSTAND THE INS
AND OUTS OF NIH AND HELP THEM
GET MENTORING IF THEY'RE IN
NEED.
WE ALSO CONDUCT OUTREACH THROUGH
SCIENTIFIC CONFERENCES AND
WORKSHOPS.
QUE PROVIDE INPUT TO YOU AS YOU
WILL SEE TODAY WHEN WE MEET.
THIS IS SOME INSTRUCTION ON THE
BREAKOUT SESSIONS.
THE PROGRAM DIRECTORS DURING THE
SESSIONS WILL SHARE WITH YOU THE
BACKGROUND AND HIGHLIGHTS OF
THEIR SPECIFIC PORTFOLIOS.
THEY WILL TALK ABOUT OTHER
EXAMPLES WHERE MEMBERS OF THE
COALITION HAVE BEEN INVOLVED IN
AUGMENTING OR DIRECTING OR
HELPING US UNDERSTAND WHAT THE
NEEDS ARE FOR THE PARTICULAR
DISEASE AND PATIENT GROUP THAT
THEY'RE ADDRESSING.
THE THINGS THAT WE HOPE TO GET
FROM THIS IS OR HELP YOU GET IS
HOW WE COULD LEVERAGE THE
RESPECTIVE RESOURCES TO ACHIEVE
OUR SHARED GOALS.
WE ALL ARE LOOKING TO GET THE
BEST SCIENCE FROM THESE
INVESTIGATORS THAT WILL ADDRESS
THE PATIENT NEEDS.
AND SOMETIMES WE'RE A LITTLE IN
PROGRAM AT NIH WE'RE A LITTLE
BIT ADRIFT FROM THAT.
SO YOUR CONNECTION HELPS US
CONNECT WITH WHAT THE ACTUAL
NEEDS OF THE PATIENTS AND THE
INDIVIDUALS AFFECTED BY THE
DISEASES ARE.
AND HOW YOU CAN MORE EFFECTIVELY
ENGAGE WITH THE PROGRAM
DIRECTORS AND HOW YOU MAY BE
ABLE TO SUPPORT AND ENGAGE WITH
INVESTIGATORS.
SOMETIMES THEY'RE ADRIFT OF WHAT
THE PATIENT VOICE IS AND WHAT
THE PATIENT NEEDS ARE.
AND IN CERTAIN CASES IT COULD BE
HELPFUL TO MEET WITH ADVOCACY
GROUPS AND UNDERSTAND WHERE THE
DISCONNECT IS AND HOW THEY MIGHT
BETTER TARGET THEIR RESEARCH TO
BENEFIT THE PATIENTS.
AND THEN ALSO AWARENESS.
THAT'S A REALLY BIG FACTOR.
BECAUSE NOT ONLY DO WE NEED THE
COALITION TO HELP MAKE THE
PUBLIC AWARE OF WHAT NIAMS IS
DOING WE NEED TO HELP MAKE THEM
WEAR OF WHAT THE ISSUES ARE WITH
THE DISEASES THAT WE ARE
ADDRESSING IN OUR INSTITUTE.
AWARENESS OF THE DISEASES AND
DISORDERS -- SOMEONE THIS
MORNING ALREADY SAID THESE ARE
NOT DISEASES THAT WILL CAUSE
IMMEDIATE DEATH BUT CAUSE
LONG-TERM DISABILITY AND QUALITY
OF LIFE IS QUITE SIGNIFICANTLY
DEGRADED WITH MANY OF THE
DISEASES THAT ARE COVERED IN
NIAMS MISSION.
WE ENCOURAGE YOU TO ACTIVELY
PARTICIPATE BECAUSE I KNOW FROM
TALKING TO YOU HERE THAT YOU ARE
STRONG ADVOCATES AND YOU ARE NOT
SHY ABOUT ASKING QUESTIONS AND
EVEN SOME HARD QUESTIONS.
PLEASE FEEL FREE TO DO THAT.
THESE ARE A FEW RESOURCES WHICH
I THINK YOU HAVE PROBABLY BEEN
MADE FAMILIAR WITH BUT THE
SLIDES I THINK ARE GOING TO BE
AVAILABLE TO YOU.
HOW YOU CAN GET MORE INFORMATION
ABOUT WHAT NIAMS IS SUPPORTING
ABOUT WHAT IS GOING ON IN THE
EXTRAMURAL PROGRAM.
WE DEFINITELY HAVE WORKSHOPS AND
EVENT GOING ON AND BEING IN
TOUCH BEING ON OUR MAILING LIST
CAN BE VERY HELPFUL TO YOU.
AND FINALLY TO TRANSITION, WE
HAVE THESE BREAKOUT GROUPS AND
ANYBODY INTERESTED IN SYSTEMIC
RHEUMATIC DISEASES AND SKIN
DISEASES AREA WOULD GO TO ROOM
AB.
MUSCLE DISEASES AND BONE
DISEASES ROOM C1 AND C2 AND OA
AND ORTHOPEDIC DISORDERS, JOINT
DISEASES, G1 AND G2.
AND WE HAVE SOME WONDERFUL FOLKS
AVAILABLE.
DR. SABOTI AND MALL AND DR. CHEN
WILL BE COVERING ROOM C1 AND C
IT AND DR. TANG AND DR. LINDSEY
WILL BE DOING ROOM G1 AND G2.
I HOPE YOU'LL HAVE A REALLY
GREAT TIME WITH THIS AND LOOK
FORWARD TO HEARING FROM YOU.
NEXT SESSION I'M PLEASED TO
INTRODUCE THE NIAMS LEADER YOU
ALL KNOW THROUGH HIS LONG TIME
PARTNERSHIP WITH THE COALITION
EVIDENT WITH HIS SUPPORT AND
ENGAGEMENT DR. BOB CARTER.
DR. CARTER IS THE DEPUTY
DIRECTOR OF NIAMS A POSITION HE
HAS HELD SINCE 2008.
HE SERVED AS NIAMS' ACTING
DIRECTOR FROM DECEMBER 2018 TO
FEBRUARY 2022.
HE LEADS NUMEROUS NIAMS POLICY
INITIATIVES AND PROJECTS
INCLUDING THE NIAMS CENTERS
EVALUATION WORKING GROUP AND
CLINICAL TRIALS WORKING GROUP.
HE ALSO HELPED SPEARHEAD BACPAC
BOTH OF WHICH ARE PART OF THE
NIHs HELPING TO END ADDICTION
LONG-TERM OR HEAL INITIATIVE.
I CEDE THE FLOOR TO HIM TO
INTRODUCE THE NEXT SPEAKER.
>> LADIES AND GENTLEMEN, ISN'T
IT WONDERFUL TO ALL BE TOGETHER
AGAIN.
SINCE 2019 IT'S BEEN A LONG
PATH.
GREAT TO BE HERE.
IT'S MY PLEASURE TO INTRODUCE
OUR SPEAKER AND SHE'S HAVING AN
ISSUE WITH MUSCULOSKELETAL BUT
JOINING US VIRTUALLY BUT
HOPEFULLY SOMETHING YOU'RE
INTERESTED IN.
SHE IS THE INAUGURAL DIRECTOR OF
THE ADVANCED RESEARCH PROJECT
AGENCY FOR HEALTH OR ARPA-H.
AND SHE COMES TO THIS FROM A
BACKGROUND RELEVANT BUT WORKED
IN TWO OTHER RELATED AGENCIES
DEFENSE ADVANCE RESEARCH PROGRAM
AGENCY OR DARPA AS WELL AS THE
INTELLIGENCE RESEARCH PROJECT
ACTIVITY.
I-ARPA.
WAS AWARDED THE SUPERIOR PUBLIC
SERVICE MEDAL FOR SERVICE IN
THOSE AREAS.
SHE'S ALSO WORKED ON THE PRIVATE
SECTOR SIDE.
SHE ALSO WORKED AT THE COMPANY
OF GINKO BIO WORKS WHERE SHE HAD
POSITIONS BOTH AS AN INNOVATION
OFFICER AND BUSINESS
DEVELOPMENT.
BRIDGING BOTH ASPECTS IN.
A WONDERFUL COMBINATION.
WHAT SHE'S EXCITE ABOUT IS THE
FIRST OF THE ARPA-H PROGRAMS,
NITRO IS ARTHROARTHRITIS.
THE TASK FOR ARPA-H IS TO
IDENTIFY A CLINICAL NEED THEN
FOCUS ON WHERE A TOP-DOWN
DIRECTED INVESTMENT AND MOVING
ON AND CAN SOLVE A PROBLEM THAT
WOULD BE VERY DIFFICULT THROUGH
THE NIH PROCESS.
I THINK KNEE OSTEOARTHRITIS IS
UP THERE AS THE PERFECT MODEL TO
TAKE THE RISK, FAIL SOMETIMES
BUT GET A NEW SOLUTION TO A
PROBLEM THAT'S BEEN A LONG AND
HAS A HIGH PUBLIC IMPACT.
IT'S QUITE DIFFERENT FROM THE
NIH APPROACH WHERE WE GIVE AN
AWARD TO SOMEBODY AND IT'S QUITE
A BIT OF MONEY BUT BASICALLY
THROW THE MONEY OVER THE WALL TO
THE INSTITUTE WHERE THEY WORK
AND TELL THEM TO RECOMPETE IN
FIVE YEARS.
A DIFFERENT WAY OF DOING
BUSINESS.
I SERVED AS THE CONTACT BETWEEN
ARPA-H AND NIH AND THINGS ARE
DONE DIFFERENTLY.
AND IT ILLUSTRATES A DIFFERENT
WAY OF DOING BUSINESS OF NOT
THINKING ABOUT THE RULES, NOT
THINKING ABOUT HOW WE SUPPORT A
COMMUNITY BUT SOLVING A PROBLEM.
SO IT'S REAL EXCITING FOR ME TO
SEE IF THIS NEW APPROACH CAN
MAKE A DIFFERENCE IN
OSTEOARTHRITIS WHICH IS SUCH AN
UNMET NEED AND JUST WONDERFUL
OPPORTUNITY.
SO WITH THAT, DR. WEGGERSON.
THANK YOU.
>> I'M SORRY I COULDN'T BE THERE
TODAY.
I'M DISAPPOINTED NOT TO MAKE IT
BUT IN THE ULTIMATE IRONY
SUFFERING A HERNIATED DISK AND
I'M HAPPY TO SHARE THE MISSION
AND HOPEFULLY BE ABLE TO CONNECT
WITH YOU ALL SOON.
LET ME SHARE MY SCREEN FOR THIS
PRESENTATION.
CAN EVERYBODY SEE?
THUMBS UP?
YOU LED IN WELL TO WHAT ARPA-H
IS HERE TO DO AND I'LL SPEND
TIME ON WHAT IS THE ARPA
BUSINESS MODEL AND WHAT IS THE
FIRST PROGRAM IN THE
OSTEOARTHRITIS.
I'LL WALK YOU THROUGH THE
JOURNEY.
IT STARTED IN MARCH OF 2022 WHEN
PRESIDENT BIDEN CALLED ON
CONGRESS TO BRING THE INITIAL
APPROPRIATION FOR ARPA-H
FORWARD.
CREATING IN THE SPIRIT OF THE
BUSINESS MODEL OF DARPA TO
PURSUE AGENCY TO BRING THE MODEL
IN HOW WE SUPPORT RESEARCH AND
IN THE INTRODUCTION YOU HEARD A
LITTLE BIT ABOUT HOW WE WANT TO
DO THAT.
IT'S A PLACE WHERE WE TAKE ON
THE BIG AUDACIOUS BETS AND NOT
ALL ARE GOING TO BE SUCCESSFUL
BUT SOME OF THEM WILL BE AND WE
WANT TO BE ABLE TO HAVE A PLACE
TO LAUNCH THE PROJECTS IN A BIG
WAY.
WE'VE BEEN VERY LUCKY TO BE
LAUNCHED AS A PRESIDENTIAL
INITIATIVE AND RECEIVE THE FULL
SUPPORT OF THE ADMINISTRATION
BUT ALSO THE CONGRESS IN
LAUNCHING ARPA-H FOR THE PROMISE
OF WHAT THIS MODEL COULD BE.
SO OUR MISSION WHEN I WALKED IN
THE DOOR I'LL SAY NOTHING
EXISTED.
WE STARTED AT FIRST PRINCIPLES,
WHAT'S THE MISSION OF ARPA-H?
WE PLANTED ON THIS MISSION TO
ACCELERATE BETTER HEALTH
OUTCOMES FOR EVERYONE.
AND I WANT TO EMPHASIZE THAT
WITH THE FOCUS ON PROBLEMS FOR
SOLVING WE CAN ACCELERATE THE
TIME LINES TO BRING THE
SOLUTIONS FORWARD IN THE REAL
WORLD.
WE MEASURE OUR PROGRESS BY
BETTER HEALTH OUTCOMES.
SO IT'S NOT ACADEMIC
PUBLICATIONS, IT'S NOT FINANCIAL
RETURN ON INVESTMENT, IT'S
IMPROVING THE LIVES FOR EVERYONE
REALLY ALL AMERICANS.
WE WANT TO BE ABLE TO REACH
UNDER SERVED POPULATIONS OF
RURAL/URBAN COMMUNITIES FROM THE
BEGINNING.
A BRIEF TIME FROM THE INITIAL
CALL FOR THE PRESIDENT TO FUND
ARPA-H TO TODAY, I WAS SWORN IN
ABOUT JUST UNDER A YEAR AGO AND
WE LAUNCHED AT HOWARD UNIVERSITY
AROUND THAT TIME.
AND VERY QUICKLY STARTED TO
BUILD THE BUSINESS OPERATIONS
TEAM TO BE READY TO TAKE OUR
FIRST SOLICITATIONS IN MARCH OF
THIS YEAR.
I LIKE TO CALL IT MINIMAL VIABLE
AGENCY STATUS WHERE SOME THINGS
YOU'RE FAMILIAR WITH LIKE THE
SOLICITATION PORTAL OUR
CONTRACTING TEAM ARE NOW IN
PLACE TO MOVE VERY QUICKLY.
SOON THEREAFTER FROM THAT
ANNOUNCEMENT WE ANNOUNCED OUR
FIRST PROGRAM OR NITRO OUR
PROGRAM IN OSTEOARTHRITIS I'LL
WALK YOU THROUGH IN A MOMENT HOW
WE GOT THERE.
AND JUST LAST WEEK WE ANNOUNCED
OUR FIRST AWARD THROUGH OUR OPEN
AGENCY ANNOUNCEMENT WHICH IS A
PLACE WHERE ANYBODY CAN SUBMIT
AN IDEA FROM THE OUTSIDE WORLD
AND FROM THAT MOMENT WE
ANNOUNCED THE SOLICITATION IN
MARCH TO LAST MONTH THAT'S FIVE
MONTHS FROM PROPOSAL REVIEW TO
TRACT NEGOTIATIONS TO GETTING
THAT FUNDED.
WE ANTICIPATE THAT'S THE SLOWEST
AWARD WE WILL EVER HAVE BECAUSE
WE'RE SORT OF BUILDING THE PLANE
WHILE WE'RE FLYING IT.
WE'RE REALLY MOTIVATED BY HAVING
THESE TYPE OF SOLICITATIONS AND
AWARDS TO ALLOW US TO MOVE
QUICKLY TO THE POINT IN THE
INTRODUCTION AS WELL WE WILL NOT
ISSUE GRANTS BUT CONTRACTS
GIVING ARPA-H A SEAT AT THE
ABLE.
WE'RE LASER FOCUSSED ON SOLVING
THE PROBLEMS AND OUR PROGRAM
MANAGERS ARE ON THE HOOK TO
SOLVE THEM AND THEY'LL BE
SITTING WITH YOU HELPING YOU GET
ACROSS THE FINISH LINE.
THE WAY WE CAN DO THAT IS THE
CONGRESS HAS STOOD US UP IN A
WAY TO MOVE QUICKLY, NIMBLY AND
DECISIVELY.
WE DON'T HAVE INTRAMURAL LABS
LIKE NIH WE JUST DELVE OUT THE
FUNDS AND WE'RE INDEPENDENT FROM
NIH.
WE'RE ESTABLISHED WITHIN NIH AND
WE CAN SHARE EXPERTISE AND GAPS
IN CHALLENGES BUT ULTIMATELY WE
CAN MOVE OUT INDEPENDENTLY ON
FUNDING WITH FEEDBACK FROM THE
TEAMS.
I REPORT DIRECTLY TO SECRETARY
BECERRA AND TO BE ABLE TO HAVE
OUR OWN PATH FORWARD WHERE WITH
CAN PURSUE PROJECTS NOT LINKED
TO A SPECIFIC DISEASE OR
TECHNOLOGY.
UNIQUE TO ARPA-H WE HAVE
COMPLETE FREEDOM WITH THE
DOLLARS WHERE WE'LL SPEND THEM
AND WANT TO FOCUS ON WHERE WE
THINK WE CAN HAVE THE GREATEST
IMPACT AND A BOTTOM'S UP
APPROACH AND RATHER THAN SET A
TOP-DOWN AGENDA SAYING THESE ARE
THINGS WE'LL FUND WE HAVE
PROGRAM MANAGERS IDENTIFIED IN
THE COMMUNITY THAT BRING NOT
ONLY THEIR C.V. BUT BIG IDEA
THEY WANT TO SOLVE.
IN THE CASE OF NITRO WHICH ENDED
UP BEING OUR FIRST PROGRAM WHICH
WAS A COINCIDENCE WE WEREN'T
TARGETING THIS TO START AND A
SURGEON SAID I DID THE MATH AND
I CAN HELP THE AVERAGE SURGEON
CAN HELP MAYBE 1,000, 2,000
PATIENTS BUT I WANT TO COME TO
ARPA-H AND REVERSE
OSTEOARTHRITIS AND HAVE JOINTS
HEAL THEMSELVES AND I CAN HAVE A
GREATER IMPACT.
WE HIRED HIM ON WITH HIS VISION
AND BROUGHT HIM ON FOR A TERM
LIMIT OF THREE YEARS.
HE'S GOT THREE YEARS TO HIT THE
GROUND RUNG AND GET THE PROJECT
STARTED AND THERE'S A LOT OF
URGENCY ASSOCIATED WITH THAT.
EVERY TWO WEEKS THAT GOES BY 1%
OF HIS TIME IS UP.
WE WANT TO MAKE SURE HE'S WELL
RESOURCES.
I HOPE THAT GIVES YOU THE SPIRIT
OF HOW WE APPROACH THE
CHALLENGES.
THE SECOND PROGRAM IS A PROGRAM
MANAGER AND FINDING THE MISSION
AND DRIVE TO SOLVE THE SPECIFIC
PROBLEM IN HEALTH.
WE'RE HERE TO ACCELERATE THE
HEALTH OUTCOMES FOR THE ENTIRE
ECO SYSTEM.
WE SERVE OUR CUSTOMERS WHO ARE
THE GENERAL PUBLIC, HEALTH CARE
PROVIDERS, PATIENT GROUPS.
I KNOW THERE'S MANY THERE IN THE
ROOM TODAY THAT OUR PROGRAM
MANAGERS HAVE BEEN ENGAGING WITH
FROM ACADEMIA AND INDUSTRY AND
STAKEHOLDER AND THE NIH IS A
STAKEHOLDER AND FEDERAL PARTNERS
LIKE FDA AND CNS ARE REQUIRED TO
HELP US GET ACROSS THE FINISH
LINE AND REGULATION AND
REIMBURSEMENT ARE KEY PART OF
THIS AND WE WANT TO MAKE SURE
WE'RE ADDRESSING THIS UP FRONT
AND I'LL TALK ON THE BACK END
ABOUT THE MARKETPLACE AND
INVESTORS AND HOW WE'RE REALLY
WORKING HARD TO CREATE A
MARKETPLACE THAT CAN BE IN
RECEIPT OF ARPA-H SOLUTIONS.
SO THIS AA LITTLE BIT OF A
REFLECTION ON DARPA WHERE
SUCCESSES IN DARPA WERE FROM THE
DEFENSE DEPEND AND DOD WILL PAY
FOR YOUR SOLUTIONS IN THIS ECO
SYSTEM.
HHS IS NOT THE SAME AS DOD.
WE HAVE A FEDERATED HEALTH CARE
SYSTEM.
FINDING THE SOLUTIONS AND
TRANSITIONING THINGS INTO THE
REAL WORLD IS A MUCH BIGGER
CHALLENGE AND WE KNOW THAT WE
REALLY NEED TO ADDRESS THAT SO
THAT INNOVATION GETS OUT OF THE
LAB AND INTO THE REAL WORLD.
WE'RE GOING TO HAVE TO HELP
FACILITATE THAT AND GREECE T
THE -- GREASE THE SKIDS ALONG
THE WAY.
AND THE PROGRAM SHOULD BE
DYNAMIC SO IF WE HAVE THIS
CONVERSATION THREE YEARS FROM
NOW WE SHOULD BE TALKING ABOUT
COMPLETELY DIFFERENT PROGRAMS
BECAUSE YOU HIRED NEW PROGRAMS
AND LAUNCHED NEW PROGRAMS.
THEY COME WITH THEIR INITIAL
IDEA AND WITH ENGAGEMENT WITH
THE COMMUNITY AND OTHER STAKE
HOLDERS TO IDENTIFY OTHER GAPS
ARPA-H CAN FILL.
FOR US SUCCESS IS WHEN THE
PROJECTS MAKE IT OUT OF ARPA-H
AND INTO THE REAL WORLD.
WE'RE NOT A FOREVER FUNDER.
WE'RE HERE TO BE A TRANSACTION
TO TAKE SOMETHING COMPLETELY
UNCERTAIN AND DEMONSTRATE SOME
DEGREE OF CERTAINTY THAT
SOMETHING IS POSSIBLE AS WE MOVE
FORWARD TO DERISK IT FOR THE
INVESTORS.
ONE OF THE WAYS IS THROUGH THE
DESIGN OF OUR PROGRAMS.
EVERY PROGRAM MANAGER AT DARPA
HAS A COMMON LANGUAGE CALLED THE
HEILMEIER PROGRAM AND IT WAS
ANSWERED IN THE INITIAL
APPLICATION WHEN THE PROGRAM WAS
PITCHED THESE QUESTIONS SIMPLY
PUT WHAT ARE YOU TRYING TO DO
AND WHAT'S THE PROBLEM YOUR
TRYING TO SOLVE AND WHAT'S IT
DONE TODAY AND WHAT'S THE GAME
CHANGER YOU'LL BRING FORWARD?
WHAT THE RISKS?
HOW LONG WILL IT TAKE AND WHAT
ARE THE MIDTERM EXAMS FOR
SUCCESS?
WE KNOW SOME WILL BE BIG LEAPS
IN ADVANCES AND THAT MAY NOT
HAPPEN FOR FIVE TO 10 YEARS AND
WHAT WILL HAPPEN TO KNOW WE'RE
ON TRACK AND MEASURE THE
PROGRESS.
WE'RE NOT THROWING THE MONEY
OVER THE FENCE WE'RE THERE WITH
A FEW.
IN BLUE WE HIGHLIGHTED HOW ARE
WE ADDRESSING THE ALL-AMERICAN
PART OF OUR MANDATE.
IF WE SUCCEED WHAT THE HEALTH
OUTCOMES THAT WE'RE GOING TO
CHANGE AND HOW ARE WE GOING TO
ASSURE EQUITABLE ACCESS TO COST
ACCESSIBILITY AND EVEN USER
EXPERIENCE IS ADDRESSED AND HOW
ARE WE BUILDING TRUST WITH
COMMUNITIES AS WE GROW?
ON THE LEFT SIDE IS AN IMAGE
FROM LAST FRIDAY A SYMPOSIUM IN
THE DEVELOPMENT OF THE PROGRAM
DEEP ENGAGEMENT WITH FDA BEFORE
WE HAVE A PRODUCT OR TECHNOLOGY
OR GROUPS FUNDED.
WE MAPPED OUT THE END TO END
PATH TO SEE WHAT ARE MY BLOCKERS
THAT WILL STOP ME FROM BRINGING
THIS INTO THE REAL WORLD AND FDA
KNOWLEDGE THEY HAVEN'T AGREED ON
AN ANIMAL MODEL FOR
OSTEOARTHRITIS APPROPRIATE FOR
OUR PROGRAM.
EVEN BEFORE WE SELECT THE TEAM
IN COLLABORATION WITH FDA SOME
FOLKS IN THE ROOM ATTENDED THIS
FRIDAY.
WHAT'S AN ANIMAL MODEL WE CAN
AGREE ON SO WE'RE READY AND
READY FOR PRECLINICAL TRIALS FDA
BOUGHT IN.
THESE ARE THE TYPES OF THINGS WE
HOPE YOU WANT TO PARTNER WITH
ARPA-H ON AND THIS IS A BENEFIT
NOR ENTIRE COMMUNITY AND NOT
JUST ARPA-H.
I WILL ALSO NOTE THIS ALLOWS US
TO EXERCISE ONE OF OUR
AUTHORITIES AND REIMBURSE THEM
DIRECTLY FOR SERVICES AND THIS
IS ONE CASE TO BRING IN FDA
COUNTERPARTS INTO THE ROOM.
WE'RE ALSO REALLY INTERESTED IN
EXPLORING PERHAPS ACCELERATED
APPROVAL PATHWAYS AND
INCENTIVIZE PARTNERSHIPS BECAUSE
FDA CAN INCENTIVIZE THROUGH SOME
REGULATORY PRIORITIZATION AND
WE'RE EXPLORING WHAT THAT MIGHT
LOOK LIKE.
WALKING YOU THROUGH WHAT LOOKS
LIKE ONCE HAVE YOU THE
HEILMEIRER QUESTIONS DEFINED AND
WE LAUNCH THE PROGRAM LIKE NITRO
AND THEY SELECT THE TEAM OF
SOLVERS AND WE LOOK TO THE
COMMUNITY FOR A SOLUTION.
WE WON'T PRESCRIBE A SOLUTION.
WE WANT TO HEAR THE DIFFERENT
IDEAS AND WE'LL PUT SOME HEAD TO
HEAD AND WE'RE WILLING TO
EXPLORE THOSE SIDE BY SIDE.
IT'S THE JOB OF THE PROGRAM
MANAGER TO THEN LOOK AT THOSE
MILESTONES SET OVER THE COURSE
OF A THREE, FOUR, FIVE-YEAR
PROGRAM AND SEE WHICH PERFORMER
TEAMS ARE REACHING THEIR GOALS
AND FOR THOSE THAT AREN'T, WE
MAY MAKE A DECISION TO PIVOT
RESOURCES AND NOT FUND THE OUT
YEARS OF A PROJECT.
WHAT'S IMPORTANT FOR THE PROGRAM
MANAGER IS GETTING TO A SOLUTION
NOT FUNDING EVERY GROUP ACROSS
THE FINISH LINE.
THERE'S A LOT OF ACTIVE PROGRAM
MANAGEMENT SO IT'S NOT A
SURPRISE TO ANYBODY IF FUNDING
ENDS BUT IT'S AN IMPORTANT POINT
OF WORKING WITH ARPA-H IMPORTANT
FOR THOSE THAT WANT TO WORK WITH
US TO BE AWARE OF.
AND LAST BUT NOT LEAST
TRANSITION.
SO GETTING THIS INTO THE REAL
WORLD SO SOMEBODY ELSE CAN PICK
IT UP AND FUND IT TO RUN THE
CLINICAL TRIAL OR COMMERCIALIZE
IS AN IMPORTANT PART OF WHAT WE
DO.
WE DEDICATED AN ENTIRE OFFICE
FOR A PROJECT ACCELERATED
TRANSITION INNOVATION OFFICE.
WE KNOW HOW HARD IT IS TO GET
THINGS IN THE REAL WORLD AND
WE'RE WORKING WITH PROGRAM
MANAGERS TO DO A MARKET
ASSESSMENT.
IT'S OKAY IF A MARKETPLACE
EXISTS BUT I WANT TO KNOW THAT
UP FRONT AND UNDERSTAND THE
GAPS.
WE ALSO DESIGN OUR PROGRAMS WITH
HUMAN CENTERED DESIGN IN MIND.
ALL OF US HAVE HAD INTERACTION
WITH THE HEALTH CARE SECTOR.
NOT ALL HAVE BEEN GOOD.
IMAGINE IF WE GET INPUT BEFORE
WE LAUNCH PROGRAMS.
WHAT'S THE COMMUNITY ACTUALLY
REALLY WANT.
WHAT'S IMPORTANT FOR FOLLOW UP.
WE KNOW WITH MAMMOGRAM THERE'S
200,000 PREVENTIBLE CANCER
DEATHS EVERY YEAR IF THEY GET A
POSITIVE MAMMOGRAM THEY
FOLLOW-UP WITH A BIOPSY.
HOW DO WE CREATE A NEW DESIGN
THAT ALLOWS FOR THE LOOPS TO BE
CLOSED.
IT'S AT THE FRONT OF MIND FOR
ALL OF OUR PROGRAM MANAGERS TO
THE END OF A PROGRAM GETTING IT
OUT TO THE REAL WORLD.
WHILE PROGRAMS ARE BOTTOM-UP AND
BEFORE I CAME TO ARPA-H I WANTED
TO ARTICULATE FOUR AREAS OF
HEALTH I THOUGHT WERE RIPE FOR
ADDITIONAL FUNDING BY ARPA-H TO
CREATE THE ASYMMETRICAL BENEFITS
AND THEY'RE NOW OUR MISSION
OFFICE UNDER A MISSION OFFICE
DIRECTOR.
THE FIRST IS HEALTH SCIENCE
FUTURES.
AND TOOLS AND TECHNOLOGIES THAT
ARE DISEASE AGNOSTIC TO ADVANCE
THE STATE OF THE ART.
WE THINK OF THE SKUNK WORKS OF
LIKE WHAT'S THE NEW mRNA
PLATFORM WE CAN DEMONSTRATE
ACROSS SEVERAL DIFFERENT
DISEASES.
DR. JENKINS IS THE DIRECTOR OF
THIS OFFICE AND WE'RE HIRING
PROGRAM MANAGERS IN ALL THE
OFFICES I SHOULD SAY.
SCALABLE SOLUTIONS IS REACHING
EVERYONE QUICKLY WHETHER IT'S
MANUFACTURING OR GETTING THINGS
TO PEOPLE IN THEIR HOMES IN
RURAL SETTINGS WHERE THERE'S
UNDER SERVED COMMUNITIES.
HOW ARE THEY BRIDGING THE LAST
MILE GAP AND WHAT'S THE
INNOVATION I NEED TO SEE.
MAYBE SOMEBODY IN THE ROOM WOULD
BE INTERESTED IN PURSUING A ROLE
AS A SCALABLE SOLUTIONS
DIRECTOR.
WE'D LOVE TO HEAR FROM YOU.
AND KEEPING PEOPLE FROM BECOMING
PATIENTS IN THE FIRST PLACE.
WHAT'S THE DIAGNOSTIC DETECTION,
BEHAVIORAL, SOCIAL SCIENCE
PROGRAMS WE COULD BE FUNDING.
ONE OF OUR PROGRAM MANAGERS SAID
THIS IS THE OFFICE WHERE YOU DIE
HEALTHY.
LIVE YOUR HEALTHIEST LIFE AND
DIE AT THE END.
ALL ALONG THE WAY YOU HAVE YOUR
HEALTHIEST LIFE SPAN AND WE WANT
TO PROMOTE THE LONGEVITY IN
HEALTH AS LONG AS POSSIBLE AND
LAST BUT NOT LEAST, RESILIENT
SYSTEMS.
THE OTHER OFFICES ARE THE
PRODUCT INNOVATION AND RESILIENT
SYSTEMS IS MORE OF A SYSTEMS
LEVEL.
WHAT IS THE INTEGRATED DATA
COLLECTED AND WHAT IS NETWORK
CAPABILITY WE CAN ESTABLISH
THROUGH OUR ARPA-H THAT WE CAN
DOUBLE DOWN IN BETWEEN PANDEMICS
PRIOR TO A CLIMATE CRISIS TO
MAKE SURE THAT WE CAN RESPOND
MOVING FORWARD.
AND SO I WILL DO A LITTLE BIT OF
A DEEP DIVE ON THE NITRO
PROGRAM.
I'M A GENETICISTS NOT AN EXPERT
IN OSTEOARTHRITIS AND HOPE TO DO
THE PROGRAM JUSTICE WITH THE
GOALS WE HOPE TO PURSUE.
SO IN THIS FIRST PROGRAM, WE
WANT TO CREATE THE VISION OF THE
FUTURE WHERE WE CAN
REVOLUTIONIZE THE CARE ALGORITHM
FOR OSTEOARTHRITIS AND I KNOW
THERE'S A LOT OF PAIN AND I'M
FEELING SOME TODAY AND REPEAT
PAIN AND COMMUNITIES DON'T HAVE
ACCESS AND THIS IS WHAT ROSS SET
OUT ADDRESS.
IT'S A TECHNICAL PROGRAM
IDENTIFIED THREE TECHNICAL AREAS
OVER THE COURSE OF THE PROGRAM
WHERE HE'D LIKE TO SEE AN
ADVANCE OVER THE STATE OF THE
ART.
THE FIRST IS A NEEDLE-BASED
NON-INVASIVE BONE REGENERATION
PROJECT IN TA1.
TECHNOLOGY AREA 2 IS LOOKING AT
NON-INVASIVE CARTILAGE
REGENERATION AND TA IS ALL-G
ALL-GENIC -- ALLOGENEIC
AUTOGENOUS NON-IMMUNOGENIC
LOAD-BEARING AND TOTAL
REPLACEMENT JOINTS AND THERE'S A
TREMENDOUS DIFFERENCE IN THE
HEALTH OUTCOMES AND THIS IS WHAT
THEY'LL HAVE TO PURSUE TO LEAN
INTO STACKING THE DECK FOR A
BREAKTHROUGH.
SO THERE BE A VARIETY OF
APPROACH PURSUED BUT MEASURING
THE ELIMINATION OF THE NEED FOR
REPEAT SURGERY.
LOOKING ACROSS THE ENTIRE
DISEASE SPECTRUM FROM EARLY
DISEASE TO LATE STAGE AND
LOOKING AT DEPARTURES FROM
FORMER USES AND MAKE SURE IT'S
REPRESENTATIVE OF THE TRUE
DEMOGRAPHIC OF THE DISEASE.
WE KNOW IT'S GOING TO BE HARD
BUT SOMETHING WE'RE PASSIONATE
ABOUT AND COMMITTED TO PROBLEM
SOLVING TO GET US ACROSS THE
FINISH LINE.
AND I ENCOURAGE ALL OF YOU TO GO
LOOK AT THE METRICS FOR THE
NITRO PROGRAM.
THERE'S A DEEP AGENCY
ANNOUNCEMENT WALKING YOU THROUGH
THE REQUIREMENTS OF THE COURSE
OF THE PROGRAM.
AND THE WHY THE EQUITY
REQUIREMENTS UNDERSTANDING THE
KEY PERFORMANCE INDICATORS WE
WANT TO TRACK AS PART OF THE
PROGRAM AND SOME LISTENING
SESSIONS MAKING SURE AS WE
DEVELOP THE CAPABILITIES WE'RE
NOT PRESCRIBE ING -- PRESCRIBING
BUT WORKING IN PARTNERSHIP TO
AND THERE'S GOING TO BE A ROAD
MAP CREATED BY PERFORMERS
THEY'LL HAVE TO FOLLOW OVER THE
COURSE OF EFFORT MOVING FORWARD.
AND IN PHASE 2 I'LL SPEAK AT THE
END OF THIS MISSION DECK A
LITTLE BIT ABOUT ARPA-H WHICH WE
WANT TO USE NOT ONLY FOR THE
NITRO PROGRAM AND CLINICAL
TRIALS TO ACCELERATE ENROLLMENT
IN CLINICAL TRIALS AND TAKE
ADVANTAGE OF THE SCALE OF A
NATIONWIDE NETWORK.
AND BRIEFLY THE OSTEOARTHRITIS
MODEL SYMPOSIUM WHICH TOOK PLACE
JUST LAST FRIDAY AND THINKING
ABOUT WHAT IS THE LARGE ANIMAL
MODEL THAT'S GOING TO BE
APPROPRIATE FOR THE PROGRAM WE
CAN LOCK IN AND DECREASE THE
PRICE OF THERAPEUTICS.
FOR THOSE THAT PARTICIPATED I
WANTED TO SHARE A LITTLE BIT OF
THE FEEDBACK.
EVEN BEFORE THE SYMPOSIUM WE RAN
A SURVEY.
THIS IS THE FIRST TIME WE'VE
DONE THESE AT ARPA-H.
THERE'S A LOT OF FIRSTS AND WE
WANT TO BRING VALUE TO THE
SCIENTIFIC COMMUNITY.
STARTING WITH WHAT IS YOUR
PREFERRED ANIMAL MODEL BEFORE
THE EVENT AND POST-SURVEY HAS
YOUR LARGE ANIMAL MODEL CHANGED?
IT'S WIDELY SEEN IT'S NOT
CHANGED BUT THERE'S MOVEMENT IN
UNDERSTANDING WHAT WILL BE
REQUIRED FROM FDA.
I UNDERSTAND THERE'S QUITE A BIT
OF FOLLOW UP THAT WILL BE
AVAILABLE TO THE GENERAL PUBLIC
AS WE CONTINUE THE CONVERSATION
ON THE ANIMAL MODEL MOVING
FORWARD.
IMPORTANTLY, I THINK A GOOD
SIGNAL FOR US IS THE QUESTION DO
YOU HAVE A BETTER UNDERSTANDING
OF THE REGULATORY REQUIREMENT?
THE RESOUNDING, YES, IS A ROLE
ARPA-H CAN PLAY IN PARTNERSHIP
WITH OUR STAKEHOLDERS AT FDA AND
A FOLLOW-UP ON THE PAYER MODEL.
IT'S NOT ALWAYS CONNECTED WITH
HOW YOU GET SOMETHING REGULATED
AND OUT THE DOOR.
WE'RE HAPPY TO CONTRIBUTE IN
THIS WAY MOVING FORWARD.
JUST TO ROUND IT OUT, THE
JOURNEY OF A PROGRAM AT ARPA-H
STARTS WITH THE PROGRAM MANAGER
AND THE PROBLEM THEY'VE
IDENTIFIED.
THE SOLICITATION, THE
APPLICATIONS ARE RECEIVED AND
WHERE NITRO IS IN EVALUATING
THE FINAL PROPOSALS AND IN THE
NEXT MONTHS WILL MAKE A
SELECTION OF TEAMS TO MOVE
FORWARD AS A PERFORMERS TO
UNDERSTAND THE NITRO RAM.
-- PROGRAM.
I HOPE SOME OF YOU APPLIED AND
STAY TUNED FOR THE ANNOUNCEMENT.
THIS ISN'T A ONE AND DONE.
WE ACKNOWLEDGE THE CHALLENGE
THAT MANY AMERICANS LIVE VERY
FAR AWAY FROM HEALTH CARE
CENTERS AND WE ALSO ACKNOWLEDGE
THAT BIO TECH AND INNOVATION IS
CLUSTERED IN A FEW PARTS OF OUR
COUNTRY AND NOT OFTEN CO-LOCATED
WHERE PATIENTS REALLY ARE.
AND SO NUMEROUS DISEASES AS A
RESULT DISPROPORTIONATELY IMPACT
THIS AND THERE'S A NOD TO
HISTORY OF THE ARPA NET AT DARPA
WHEN THEY LAUNCH THE PROGRAM
THAT BECAME THE INTERNET WAS A
FOUR-NODE NETWORK
I DON'T KNOW HOW MANY NODES THE
INTERNET HAS TODAY BUT CERTAINLY
MANY MORE THAN FOUR NODES.
WE'RE STARTING WITH THREE NODES
AND THIS IS LEGISLATIVELY
DETERMINED CONGRESS GAVE US
THREE SITES.
WE WANT TO LEAN INTO THE THREE
GEOGRAPHIES AND BUILD A 50-STATE
HUB AND SPOKE NETWORK.
THREE HUBS WILL UNLOCK CERTAIN
KEY CAPABILITIES WE NEED TO
TRANSITION THINGS TO THE REAL
WORLD.
SO FIRST WE'LL HAVE A
STAKEHOLDER AND OPERATIONS HUB
IN THE WASHINGTON, D.C. REGION,
EXACT ADDRESS TO BE DETERMINED
BUT IT WILL KEEP US CLOSE TO CMS
TO OUR FDA PARTNERS, HRSA AND
OTHERS WE KNOW WILL HELP US GET
TECHNOLOGY TO THE REAL WORLD AND
OTHER TWO WE'LL ANNOUNCE NEXT
WEEK WHERE THE HUBS LAND.
A CUSTOMER EXPERIENCE HUB
FOCUSSED ON THE PEOPLE WE'RE
INTENDED TO SERVE.
HOW DO WE ENGAGE COMMUNITIES AND
ENROLL COMMUNITIES IN CLINICAL
TRIALS.
HOW DO WE PUSH OUT THE BEST
INNOVATION AS QUICKLY AS
POSSIBLE AND A PLACE FOR
MANAGERS TO TRY TO CREATE NEW
SOLUTIONS TO ARPA-H INVESTMENT
MOVING FORWARD AND LAST BUT NOT
LEAST OUR INVESTOR CLAFT
CATALYST HUB.
ARPA-H IS NOT A LONG-TERM FUNDER
AND WE WORK TO FUND PROJECTS
POST ARPA-H BUT THE MAJORITY OF
ARPA-H PROGRAMS WILL GO IN THE
PRIVATE SECTOR AND BE
COMMERCIALIZED.
SO WE WANT TO START TO CURATE
THE GROUP OF INVESTORS WE CAN
START TO UNDERSTAND WHAT DO WE
NEED TO DERISK FOR THEM TO PICK
UP FUNDING ON THE BACK END?
WE'RE ALSO FOCUSSING ON
INVESTORS NOT JUST LOOK FOR
FINANCIAL RETURN ON INVESTMENT
BUTTING IN COMMUNITIES AND
REGIONAL INVESTMENT AND GROWING
SUSTAINED LEVEL HEALTH CARE AND
IT'S A TOP PRIORITY FOR US AS WE
LAUNCH THE ARPA-H NET H.
I SHARED A LOT OF THIS
INFORMATION WITH YOU IN THE
PRIOR SLIDE.
I WANTED TO HAMMER HOME THAT WE
DON'T WANT TO DEVELOP SCIENCE IN
THE ABSENCE OF THE CUSTOMER.
SO THIS IS SUCH AN INTEGRAL PART
OF WHAT WE DO AT ARPA-H.
AND A CALL TO ACTION WITH THE
LAST SLIDE AND THEN HAPPY TO
TAKE QUESTIONS.
SUBMIT YOUR IDEA AND OUR PROGRAM
MANAGERS LAUNCHED PROGRAMS LIKE
NITRO BUT WE HAVE AN OPEN DOOR
FOR ANY IDEA YOU MAY WANT TO
PITCH US AND WE SAY IT'S THE
THREE-PAGER.
SHARE YOUR IDEA WITH US AND WE
WANT TO HAVE A CONVERSATION
AROUND IT.
MAYBE YOU WANT TO BECOME A
PROGRAM MANAGER AND MAY BE AN
AREA YOU'RE PASSIONATE ABOUT
SOLVING.
WE'LL PURSUE AN AREA OF SCIENCE
IF WE HAVE THE CHAMPION THAT
PROGRAM MANAGER THAT WANTS TO DO
THIS SIMILAR TO WHAT ROSS DID.
AND WE LAUNCHED A NEWSLETTER AND
YOU CAN SIGN UP AND HEAR THE
LATEST AND GREATEST.
TO THE NIAMS TEAM THANK YOU FOR
INVITING ARPA-H TO PRESENT IN
THE SESSION TO SHARE A LITTLE
BIT ABOUT THE WORK WE'RE DOING
ALIGNED WITH YOUR INTERESTS AND
I'M EXCITED TO HEAR THE
QUESTIONS.
THANK YOU.
>> THANK YOU.
IT'S THRILLING TO SEE THE
AMBITI
AMBITIOUS.
WE WISH YOU THE BEST.
WON'T BE WONDERFUL FIVE YEARS
FROM NOW WITH A DIFFERENT VIEW
OF OSTEOARTHRITIS WITH THE
EQUITY AND POPULATION HEALTH
ASPECTS.
THAT'S A TALL CHALLENGE BUT
THERE ARE THOSE IN THE ROOM WHO
LIVE THEIR LIFE TO ADDRESS THIS
PROBLEM SO IT'S EXCITING TIMES.
I'M HOPING TO SEE SOME HANDS.
MIKE.
>> PEDIATRIC DERMATOLOGY.
WHAT HAPPENS TO THE PROGRAM
MANAGERS AFTER THE THREE YEARS?
SOME OF THE PROMISING MINDS WHO
WOULD DO THIS STUFF MAY BALK AT
LEAVING THEIR JOB FOR THREE
YEARS AND CURIOUS ON THOUGHTS ON
THAT AND HOW YOU'LL CULTIVATE
THAT OVER TIME.
>> GREAT.
I THINK I HEARD THE QUESTION,
WHAT HAPPENS TO A PROGRAM
MANAGER AFTER THREE YEARS.
YOU TURN INTO A PUMPKIN AND THEN
WHAT?
BY STATUTE IT'S THREE YEARS BUT
IT'S RENEWABLE UP TO ANOTHER
THREE YEARS IF CAN AGREE TO
TERMS AND SIX YEARS IS IT AND
WE'RE LOOKING FOR DIVERSITY AND
DEMOGRAPHICS AND GEOGRAPHY.
WE'RE NOT MAKING ANYBODY MOVE SO
TO YOUR POINT THIS IS LEAVING
YOUR CAREER POTENTIALLY AND WE
DON'T WANT YOU TO HAVE TO MOVE
YOUR WHOLE FAMILY SO YOU DON'T
HAVE TO MOVE BUT MUST BE WILLING
TO TRAVEL AND WE'RE RECRUITING
FOLKS THAT HAVE GOVERNMENT
EXPERIENCE OR ACADEMIC
EXPERIENCE OR INDUSTRY
EXPERIENCE.
THOSE ARE ALL VALID AS WE GROW
THAT.
I CAN SPEAK -- WE HAVEN'T
GRADUATED ANY PROGRAM MANAGERS
YET.
WE'RE JUST STARTING BUT IN MY
EXPERIENCE AT DARPA THIS CAN
UNLOCK A LOT OF DOORS.
YOU BECOME AS A PROGRAM MANAGER
THE FACE AND VOICE OF THE
CHALLENGE FOR ARPA-H IN OUR
CASE.
YOU START TO BUILD NETWORKS WITH
YOUR COMMUNITY AND START TO TEE
UP WHAT DO YOU WANT TO DO NEXT?
WHAT'S THE BIG PROBLEM YOU WANT
TO SOLVE?
SOMETIMES PEOPLE GO BACK.
WE CAN DO WHAT'S CALLED AN
I.P.A., INTERPERSONNEL AGREEMENT
AND YOUR UNIVERSITY MAY GIVE US
TO US ON LOAN AND YOU CAN GO
BACK TO PICK UP WHERE YOU LEFT
OFF AND NOW HAVE EXPERTISE FROM
AN ARPA-H MODEL AND UNDERSTAND
THE FEDERAL GOVERNMENT MORE AND
WORKED WITH FDA AND CMS AND HAVE
A BETTER REVIEW OF THE WHOLE ECO
SYSTEM.
THAT'S IN A WAY OTHERS DON'T
HAVE WITH THIS EXPERIENCE AND SO
ARE SO MOTIVATED TO SOLVE THIS
PROBLEM THEY DONT LOOK AT IT AS
GIVING UP A POINT IN OUR CAREER
AND IF I LAUNCH THIS ARPA-H WILL
GIVE ME A BUDGET OF $100 TO 150
MILLION TO SOLVE A PROBLEM
THAT'S BEEN A BLOCKER.
AND IT'S A HARD JOB.
YOU'RE SPRINTING FOR THREE
YEARS, RIGHT.
SO YOU HAVE TO FIND THE FOLKS
DETERMINED TO BE THE DOERS TO
UNSTICK AND SOLVE THAT PROBLEM.
>> ONE OF THE BIG ISSUES IS THE
PRESUMPTION IF YOU CAN REBUILD
CARTILAGE YOU'LL TREAT PAIN?
>> THERE'S A METRIC AS I
UNDERSTAND THAT PAIN IS A METRIC
AND REDUCTION OF PAIN IS
SOMETHING THEY'LL BE MONITORING
AS ONE OF THE HEALTH OUTCOMES.
IT WILL BE MULTIPLE HEALTH
OUTCOMES MONITORED BUT THAT'S
CERTAINLY ONE OF THEM.
[OFF-MIC]
>> OUR INITIAL APPROPRIATION WAS
$1 BILLION AND A SECOND
APPROPRIATION FOR $1.5 BILLION.
WE'RE THE ONLY OPERATING
DIVISION I'M AWARE OF THAT HAS
WHAT'S CALLED THREE-YEAR MONEY.
WE DON'T HAVE TO SPEND IT ALL BY
THE END OF THE FISCAL YEAR, SO
NEXT WEEK.
WE HAVE THREE YEARS TO SPEND THE
MONEY.
THAT ALLOWS US TO MILLION AHEAD
ON OUR PROGRAMS.
WITH ROSS ASKED FOR HIS BUDGET
FOR HIS PROJECT, THE WAY I THINK
IS YES, YOU CAN DO YOUR NITRO
PROGRAM AND HERE'S YOUR BANK
ACCOUNT.
IT'S THEN UP TO ROSS AS WE LOOKS
THROUGH THE PROPROPOSALS AND HE
THEM EVALUATE THE PROPOSALS BUT
WE DO NOT MAKE DECISIONS BY
STUDY SECTION.
HE'S A SOUL DECISION MAKER.
HE LOOKS AT THE PROPOSALS
INCLUDING A TECHNICAL STATE OF
WORK AND BUDGET.
THERE'S A DEEP NEGOTIATION BACK
AND FORTH.
WE DON'T TAKE IT AT FACE VALUE.
IT'S NEGOTIATION AROUND THE
COST.
YEAR NOT LOOKING FOR THE LOWEST
BIDDER BUT COST REALISM.
IF WE'RE EXCITED ABOUT THE
REGENERATIVE BONE PROGRAM THAT
YOU WANT TO MOVE FORWARD WITH,
YOU WANT TO MAKE SURE YOU HAVE
THE RIGHT TEAM IN PLACE YOU HAVE
ALL THE BIO REACTORS AND THEY
TEND TO BE WELL RESOURCED
PROJECTS FORWARD.
IT'S UP TO THE PROGRAM MANAGER.
FOR ME AT THE AGENCY LEVEL,
WHAT'S IMPORTANT FOR ME IS
FUNDING A DIVERSITY PORTFOLIO
AND I NEED TO DEMONSTRATE ARPA-H
IS HERE TO CATALYZE THE ECO
SYSTEM.
THE ANSWERS FROM THE ARPA-H
NITRO AND OUR SURGICAL
INTERVENTIONS.
LAST WEEK WE ANNOUNCED A
MODULATORY PROGRAM.
WE CALL IT HIGH CONTACT PROGRAM
MANAGEMENT.
THEY'RE WELL RESOURCED PROJECTS
BUT IT'S NOT THROWN OVER THE
FENCE.
WE'RE THERE WITH YOU AND IF
THINGS AREN'T WORKING OUT WE
RESERVE THE RIGHT TO PIVOT THE
FUNDS ELSEWHERE TO MAKE MASSIVE
IMPACT.
I HOPE THAT HELPS.
>> I'M WITH THE NATIONAL SCLER
ODOR -- SCLERODERMA FOUNDATION.
WHAT CAN WE DO TO HELP AND
FACILITATE THE INTERACTION SO
PATIENT VOICE IS INCLUDED IN
THAT DECISION MAKING?
>> CAN YOU REPEAT THAT QUESTION
THAT WAS A LITTLE BIT CHOPPY FOR
ME.
>> YOU TALKED ABOUT ENGAGEMENT
WITH THE GENERAL PUBLIC AND
PATIENT COMMUNITY AND US,
PATIENT ADVOCACY ORGANIZATIONS.
WHAT CAN WE DO TO HELP AND
FACILITATE THOSE DISCUSSIONS AND
ENSURE THAT PATIENT VOICE IS
INCLUDED IN YOUR DECISION
MAKING?
>> AND WE HAVE ABOUT 200 PATIENT
GROUPS AND ONE IS LETTING PEOPLE
KNOW WE'RE HERE AND WHAT WE DO
IS DIFFERENT FROM THE REST OF
THE ECO FUNDING SYSTEM.
THAT'S BEEN AN IMPORTANT
LEARNING AND YOU SAY DARPA AND
PEOPLE GET IT BUT IN A HEALTH
SYSTEM THEY DON'T KNOW ARPA-H OR
DARPA.
WE HAVE TO EXPLAIN THE BUSINESS
MODEL.
WE SPEND A LOT OF TIME ENGAGING
WITH THE VARIETY OF PATIENT
GROUPS TO GET TO KNOW US AND NOW
THAT WE'RE LAUNCHING PROGRAMS
IT'S UP TO THE PROGRAM MANAGERS
TO START TO MAKE THOSE
CONNECTIONS.
I KNOW ROSS HAS BEEN WORKING
WITH THE ROOM THERE AND THE
FOLKS THAT ARE PRESENT BUT THE
OTHER WAY WE WANT TO FACILITATE
THIS IS THROUGH ARPA NET H AND
YOU'LL SEE THE DIFFERENT GROUPS
WHEN WE ANNOUNCE THE BESPOKE.
IT WILL BE THAT SNAPSHOT AT TIME
BUT IT'S MEANT TO BE DYNAMIC
WITH PROGRAM AND WHAT I DIDN'T
TALK ABOUT IS HOW WE'RE GOING TO
DO THE INTERACTIONS.
THAT PROJECT IS EXCLUSIVELY
OTHER TRANSACTION AUTHORITIES
WHICH MEANS SEVEN DAYS TO GET
UNDER CONTRACT.
WE WANT TO BE ABLE TO FUND
COMMUNITIES TO SUPPORT SOME OF
OUR EFFORTS AND IN ROSS'S CASE
WHEN HE LAUNCHES THE CLINICAL
TRIAL HE HAS THE BUDGET TO WORK
ON THE CIVIL ENGINEERING BUT
WE'LL REFER A BUDGET FOR ARPA
NET H TO DO PATIENT OUTREACH AND
HAVE FUNDS ASSOCIATED AND WE
HEARD LOUD AND CLEAR FROM
COMMUNITIES IT'S GREAT TO BE IN
THE ROOM BUT IF YOU'RE NOT
PAYING FOR ME TO BE HERE AND MY
TIME IT MAKES IT CHALLENGING.
WE WANT TO BE RESPONSIVE BUT
WE'RE OPEN TO IDEAS.
IF ANYONE WANTS TO REACH OUT TO
IDEAS OF BEST PRACTICES OR
WHAT'S WORKED FOR YOU AND I'M
HAPPY TO SHARE THIS.
IT'S GOING TO BE LISTENING AND
LEARNING.
>> ONE LAST ONE.
>> I'M WITH THE NATIONAL
PSORIASIS FOUNDATION.
WHEN WE THINK OF HEALTH OUTCOMES
AND HOW IT ACHIEVE THEM WE GET
STRUCK AT THE TRIALS AND CERTAIN
APPROACHES TO HEALTH CARE GIVEN
YOU'RE PROGRAM IS DEDICATED TO
THINKING OUTSIDE THE BOX, HOW
RECEPTIVE ARE YOU TO DIFFERENT
APPROACHES TO DELIVERING
CLINICAL TRIAL OR HEALTH CARE
THAT MAY IMPROVE OUTCOMES
OUTSIDE THE TRADITIONAL HEALTH
CARE SYSTEM?
>> I THINK YOU WERE ASKING ABOUT
HEALTH CARE IMPACT OUTSIDE THE
SUF
U.S.
>> WE KNOW CERTAIN PATIENTS ARE
TRADITIONALLY UNDER SERVED AND
THEIR HEALTH OUTCOME WILL BE
WORSE THAN THE AVERAGE
POPULATION.
THINKING YOUR PROGRAM ARPA-H IS
SUPPOSED TO THINK OUTSIDE THE
BOX.
HOW MUCH ARE YOU THINKING OF
DIFFERENT METHODS IN ORDER TO
REACH PATIENTS TO IMPROVE THEIR
HEALTH OUTCOMES VERSUS MORE
ADDITIONAL RESEARCH CLINICAL
TRIALS APPLICATION TO THINK
ABOUT IF YOU'RE FOCUSSED ON I AM
PROVING HEALTH OUTCOMES THE
EASIEST WAY IS TO GET PATIENTS
TO LINK UP TO EXISTING HEALTH
CARE OR EXISTING HEALTH CARE
INNOVATIONS --
>> IT'S A BAD CONNECTION.
I APOLOGIZE IF I DIDN'T GET ALL
THAT.
HOW ARE WE GOING TO INFLUENCE
HEALTH CARE?
>> IF THERE'S ROOM FOR ARPA-H
WITH THESE APPROACHES OR
THINKING MORE ABOUT CLINICAL
TRIALS OR DIFFERENT TRYING TO
UNDERSTAND DISEASES RATHER THAN
DELIVERING HEALTH CARE.
>> THE ARPA-H MANDATE IS PRETTY
WIDE OPEN.
WE CAN FUND CROSS WHAT I THINK
ARE TECHNOLOGY READINESS LEVELS
ON THE BACK OF A NAPKIN AND WE
CAN FUND THE ENTIRE CONTINUUM.
WE'RE NOT HERE TO REFORM THE
HEALTH CARE SYSTEM.
WE'RE HERE TO DEMONSTRATE AND
FAVOR THE ODDS OF BREAKTHROUGHS
THAT CAN BE ADOPTED.
ARPA-H IS A NOD WE NEED TO
INTEGRATE IN THE HEALTH CARE
SYSTEM AND FIGURE OUT THE PAYER
MODELS.
WE'RE BASICALLY A TECHNOLOGY
ORIENTED ORGANIZATION.
FOUR YEARS FROM NOW WILL BE AN
INTERESTING MOMENT IN TIME
BECAUSE CONGRESS CALLED ON US TO
COME BACK IN FRONT OF THE
CONGRESS AND SAY FOUR, FIVE
YEARS IS PAST ARPA-H.
GIVE ME THE QUANTITATIVE
OUTCOMES HOW YOU IMPROVED HEALTH
CARE IN THE UNITED STATES.
THE IS HARD BECAUSE MOST ARPA-H
TIME LINES TEND TO BE 10 TO 15
YEAR TIME LINES WHERE YOU START
TO SEE THE IMPACT IN THE
COMMUNITY.
SOMETHING WE'RE THINKING ABOUT
AND THE IMMEDIATE STEPS WE CAN
DEMONSTRATE SOME OF THAT
MOVEMENT IN THE U.S. MOVING
FORWARD AND THIS IS A PASSIONATE
AREA FOR THE PRESIDENT AND FIRST
LADY.
THEY'VE TRYING TO LOOP US IN THE
CONVERSATION OF HOW DO WE CHANGE
THE WAY WE THINK OF HEALTH CARE
IN THE UNITED STATES AND CREATE
SPACES WE CAN INNOVATE AND TAKE
RISKS.
MAYBE IN A YEAR FROM NOW WE'LL
HAVE ADDITIONAL PROGRAMS THAT
START TO ADDRESS IT.
I DON'T KNOW ENOUGH TIME HAS
PASSED TO SAY HOW WE'RE GOING TO
IMPACT HEALTH CARE IN THE UNITED
STATES.
>> LET US KNOW WHEN YOU FIGURE
IT OUT.
THANK YOU, VERY MUCH.
THAT WAS WONDERFUL.
THANK YOU.
>> THANK YOU SO MUCH.
TAKE CARE, GUYS.
>>
>> THAT WAS A TERRIFIC
PRESENTATION TO KICK OFF OUR
AFTERNOON SESSIONS.
A COUPLE OF FEW HOUSEKEEPINGS
AND GUIDANCE FOR THE AFTERNOON.
WE'RE NOW GOING TO BE TRANSITION
TO POSTER SESSION WHICH I'M
EXCITE ABOUT.
IT'S ANOTHER OPPORTUNITY FOR YOU
ALL TO NETWORK AND ENGAGE WITH
EACH OTHER AND ENCOURAGE YOU TO
DO THAT AS MUCH AS POSSIBLE.
THE WAY THE POSTER SESSION'S
GOING TO WORK IS IF YOU HAVE AN
EVEN NUMBER YOU'LL BE PRESENTING
FOR THE FIRST HALF OF THE POSTER
SESSION.
INSTEAD OF 2:15 TO 2:35 WE'RE
GOING TO DO 2:20 TO 2:40 AND THE
ODD WILL PRESENT THE SECOND HALF
SO EVERYONE CAN PRESENT.
ENGAGE WITH ONE ANOTHER.
THERE'S COFFEE AND A REMINDER TO
FILL OUT THE RECOMMENDATION
FORMS THEY HELP SHAPE THE
MEETING THE CO-CHAIRS READ
THROUGH ALL OF THEM.
AS YOU GET IDEAS ABOUT WHAT
YOU'D LIKE TO HEAR OR SEE AT
THIS PLATFORM, RECORD IT ON THE
EVALUATION FORMS AND GIVE THEM
TO REGISTRATION ON YOUR WAY OUT.
ON THE TOPIC OF FEEDBACK IN THE
POSTER ROOMS WE'RE LOOKING FOR
YOUR FEEDBACK ON THE NIAMS
RESOURCES.
ANY ORGANIZATIONS RELATED TO
ARTHRITIS, LUPUS PLEASE GO TO
THE BACK TABLE WE'RE TAKE ING
YOUR FEEDBACK.
AFTER THE POSTER SESSION WE'RE
GOING TO BE GOING IMMEDIATELY
INTO THE BREAKOUT SESSION WHICH
MEANS THERE WON'T BE ANY
ANNOUNCEMENTS TO PLEASE PAY
ATTENTION TO YOUR AGENDA AND
PROMPTLY AT THE END OF THE
POSTER SESSION WHEN YOU HEAR THE
CHIME, THAT MEANS YOU GO TO YOUR
SELECTED BREAKOUT SESSIONS.
YOU SELECT THE TOPIC YOU'RE
INTERESTED IN AND YOU CAN MAKE
YOUR WAY TO THAT BREAKOUT.
WE HAVE A LOT OF SPEAKERS SIGNED
UP SO PLEASE STAY TUNED FOR
THEM.
IF YOU HAVE QUESTIONS, THERE'S
NIAMS STAFF WITH THE BLUE
RIBBONS ON AND AGAIN USE THE
OPPORTUNITY TO NETWORK, SHARE
AND LEARN FROM EACH OTHER.
WE CAN ALL MOVE OVER TO F1, F2
JUST OUTSIDE THE DOOR TO THE
RIGHT AND RIGHT AGAIN.
YOU'LL SEE A SIGN.
THANK YOU.