>> GOOD MORNING, EVERYONE.
WELCOME TO THOSE THAT TRAVELED
TO THE NIH. THANK YOU SO MUCH.
FOR THOSE THAT JOINED VIRTUALLY,
WE APPRECIATE YOU BEING WITH US.
I WILL CALL THE 114TH MEETING OF
THE NATIONAL ARTHRITIS AND
MUSCULOSKELETAL AND SKIN
DISEASES ADVISORY COUNCIL TO
ORDER. ALL MEETINGS ARE OPEN TO
THE PUBLIC AS REQUIRED BY LAW
EXCEPT REVIEW AND DISCUSSION AND
GRANT APPLICATIONS AND RELATED
INFORMATION THAT WILL BE
ADDRESSED IN CLOSED SESSION THIS
AFTERNOON.
BEFORE WE PROCEED, I WOULD LIKE
TO GO AROUND THE TABLE FOR THE
COUNCIL MEMBERS AND ANYAM STAFF
THAT ARE WITH US TODAY TO
INTRODUCE THEMSELVES.
I WILL GO TO VALENTINA.
>> HELLO. I HAVE SPENT FIF
YEARS IN SKIN LAB.
>> HI, EVERYONE I'M AT JOHN
HOPKINS IN BALTIMORE AND AD HOC
MEMBER ON CASE YEAR 23.
>> I'M THE FORMER EXECUTIVE
DIRECTOR FOR AMERICAN SOCIETY OF
BONE AND MINERAL RESEARCH.
>> I AM BETH MCNALLY AND FROM
NORTHWESTERN UNIVERSITY AND
ADULT CARDIOLOGIST AND WORKED ON
MYOPATHIC DISEASES FOR MOST OF
MY CAREER.
>> BOB CARTER AND DEPUTY
DIRECTOR OF NIAMS AND DELIGHTED
TO BE WITH YOU.
>> I'M DIRECTOR OF DIVISION OF
EXTRAMURAL ACTIVITIES AT NIAMS.
>> I'M GAIL LESTER AND DIRECTOR
OF EXTRAMURAL RESEARCH AT NIAMS
SGR I'M AN AD HOC MEMBER ON A
K23.
>> I'M HEAD OF RESEARCH AT THE
LUPUS RESEARCH ALLIANCE. THANK
YOU.
>> HELLO. I'M TAMMY AND
DERMATOLOGIST AT SOUTHWESTERN
AND [INDISCERNIBLE] IN THE SKIN
AND AN AD HOC MEMBER.
>> I'M MARIA AND RHEUMATOLOGIST
AND CLINICAL EPIDEMIOLOGISTS AT
UNIVERSITY OF TEXAS MD STANDARD.
>> NOT SURE IF THEY HAVE
MICROPHONES SHALANDA AND MARK,
CAN YOU INTRODUCE YOURSELVES?
>> GOOD MOTH R MOBLG S U -- GOO
MORNING I'M MARK AND IT SUPPORT.
>> I'M A MANAGEMENT ANALYST AT
NIAMS.
>> STEPHANIE AND MARY, SINCE YOU
ARE PRESENTING CAN YOU --
>> GOOD MORNING I'M STEPHANIE
AND CHIEF OF UN INSTITUTE AND
POLICY PLANNING IN THE
[INDISCERNIBLE] BRANCH.
>> I'M MARIA GARCIA AND DIRECTOR
OF EXTRAMURAL RESEARCH.
>> BEFORE WE TURN TO FOLKS
JOINING VIRTUALLY I WANT TO
MENTION A LARGE NUMBER OF NIAM
STAFF SITTING BEHIND US NOT AT A
MICROPHONE AND IF THEY HAD AN
OPPORTUNITY TO SPEAK UP DURING
COUNCIL I HOPE THEY WILL COME TO
THE TABLE AND INTRODUCE
THEMSELVES AS WELL.
REALLY GREAT TO HAVE THEM HERE.
WHO DO WE HAVE ONLINE?
FIRST OF ALL, ANY COUNCILMEMBERS
ONLINE?
ED, GREAT. WHY DON'T WE HAVE
ED.
>> GOOD MORNING I'M EDAND
PROFESSOR OF BIOMEDICAL
ENGINEERING AT [INDISCERNIBLE]
DEPARTMENT OF BIOMEDICAL
ENGINEERING AND RESEARCH IS IN
BIOMATERIALS AND REGENERATIVE
MEDIC
MEDICINE.
>> HI EVERYBODY I'M A
RHEUMATOLOGIST AT SAN FRANCISCO
GENERAL HOSPITAL AT USF AND
CLINICAL RESEARCHER DOING
EPIDEMIOLOGY OUTCOMES AND
CLINICAL FORMATICS.
>> CLINICAL BOB WANT TO
INTRODUCE YOURSELF.
>> GOOD MORNING EVERYBODY I'M
BOB AND CLINICAL DIRECTOR FOR
NIAMS INTRAMURAL RESEARCH
PROGRAM RESEARCH IN SPONDYLITIS
AND DOING RESEARCH THIS MORNING
AND THANK YOU.
>> COUNCIL MEMBERS ONLINE?
I SEE SUE WHO WILL SPEAK LATER
AND WE WILL HAVE HER INTRODUCE
HERSELF WHEN WE GET TO THAT
TIME. ANYBODY ELSE?
ARE IKARE YOU ABLE TO --
EXECUTIVE OFFICER ARE YOU ABLE
TO SAY HELLO. THERE WE GO.
>> I'M A NIAMS EXECUTIVE
OFFICER.
>> I WOULD LIKE TO ACKNOWLEDGE
MELINDA NELSON WHO IS HELPFUL TO
COUNCIL MEETINGS AND NOT SURE IF
SHE IS ABLE TO TURN ON HER
CAMERA BUT THANKS FOR BEING
HERE, MELINDA.
I WILL MOVE UP TO THE PODIUM AND
BEGIN THE MEETING WITH MY
REPORT.
LOGISTICS FIRST FROM
D.A.R.E..IN.
>> THANK YOU, LINDSEY. GOOD
MORNING AND WELCOME, EVERYONE.
I WILL GIVE A FEW QUICK
LOGISTICAL REMINDERS BEFORE I GO
THROUGH THE REST OF MY SKRUPT
HE
HERE.
WE HAVE CHAT FOR THE ZOOM BUT
PREFER YOU DON'T USE IT FOR
DISCUSSIONS IT IS NOT PUBLIC AND
IF YOU WANT TO SEND ME A
QUESTION YOU CAN DO THAT AND
PREFER YOU RAISE YOUR HAND IF
YOU HAVE A COMMENT TO JOIN IN
THE CONVERSATION AND FOR THOSE
IN THE ROOM WE CAN ONLY HAVE
FOUR MICROPHONES ON AT ONCE SO
JUST FY I. TURN IT OFF IF YOU
ARE NOT USING IT.
AS I MENTIONED, THERE IS A
NUMBER OF STAFF OR YOU HAVE SEEN
A NUMBER OF STAFF ARE
PARTICIPATING VIA ZOOM AND LARGE
NUMBER OF THE PUBLIC WATCHING
VIA LIVE VIDEO CAST AND IF STAFF
MEMBERS WATCHING VIDEO CAST WITH
AN URGENT COMMENT OR QUESTION
YOU CAN E-MAIL SHALANDA
WELLINGTON AT NIH.GOV WHO CAN
REFER THE QUESTION TO ME AND
ENCOURAGE VIEWERS TO SUBMIT
COMMENTS IN WRITING 15 DAYS UP
TO THE MEETING AND COMMENTS SENT
TO NIAMS NIAMS.
AGAIN, ALSO YOU CAN SEE THIS ON
OUR WEBSITE.
ANYONE ON THE ZOOM CALL MAY
SPEAK AND MUTE YOUR LINE WHEN
NOT SPEAKING AND PREFER YOU TO
RAISE YOUR HAND AND WE WILL CALL
ON YOU AND SHELL AN DAH WILL
HELP WITH THE MUTING AND
UNMUTING WHEN WE ARE TAKING
VOTES AND ASK COUNSELORS AS MUCH
AS POSSIBLE TO KEEP YOUR VIDEO
ON DURING THE DISCUSSIONS EXCEPT
DURING PRESENTATIONS TO ASK YOU
TO TURN YOUR VIDEO OFF.
IF YOU NEED COMPUTER SUPPORT OR
HELP WITH ELECTRONIC COUNCIL
BOOK DURING THE MEETING YOU CAN
CONTACT SHELL AN DAH AND NOTICE
OF THIS MEETING IS PUBLISHED IN
THE FEDERAL REGISTER AND ARE
REQUIRED TO ABSENT THEMSELVES
DURING REVIEW OF APPLICATIONS IF
PRESENCE WOULD APPEAR TO
CONSTITUTE A CONFLICT OF
INTEREST AND WOULD LIKE TO
REMIND YOU ABOUT UPCOMING
CONFLICT OF INTEREST REPORTING
DATES AND HAD ONE IN FEBRUARY
AND IN JUNE AND OCTOBER AND
RECEIVING A REQUEST MEEZ RESPOND
WITHIN 30 DAYS TO PARTICIPATE IN
COUNCIL-RELATED ACTIVITIES AND
FURTHER INFORMATION REGARDING
CONFLICT OF INTEREST REGULATIONS
ARE INCLUDED IN THE ELECTRONIC
COUNCIL BOOK THAT YOU CAN CHECK
THERE.
I WOULD LIKE TO TURN TO THE
CONSIDERATION OF THE MINUTES AND
WE ARE GOING TO TAKE A VOTE AND
COUNCIL MEMBERS ON ZOOM, PLEASE
RAISE YOUR HAND AS NECESSARY.
YOU HAVE SEEN MINUTES ON JANUARY
30TH 2024 COUNCIL MEETING WERE
POSTED IN ELECTRONIC COUNCIL
BOOK AND DOES ANYONE HAVE
COMMENTS OR CORRECTIONS?
HEARING AND SEEING NONE I WILL
MOVE WE APPROVE THE JANUARY 30TH
COUNCIL MINUTES.
DO I HAVE A SECOND?
I HAVE A SECOND.
THANK YOU.
I ASK ALL THOSE OPPOSED TO THE
MOTION TO PLEASE RAISE THEIR
HAND OR SAY SOMETHING IF YOU ARE
OPPOSED WITH THE APPROVAL.
SEEING NO OBJECTIONS, THE MOTION
PASSES.
THANK YOU VERY MUCH.
NOW I WOULD LIKE TO REMIND YOU
ABOUT DATES OF FUTURE COUNCIL
MEETINGS AND THEY ARE LISTED IN
ELECTRONIC COUNCIL BOOK AND NEXT
MEETING IS SEPTEMBER 17TH THAT
IS A HYBRID MEETING AND GOING
FORWARD WE PLAN TO HOLD THE
SPRING MEETING THAT IS THIS
MEETING IN PERSON AND THE FALL
MEETING IS SEPTEMBER MEETING
THAT IS HYBRID AND WINTER
MEETING WILL BE FULLY VIRTUAL
AND WE VERY MUCH APPRECIATE YOUR
PARTICIPATION AND MAKE SURE
DATES I WILL READ OFF NOW ARE ON
YOUR CALENDAR SEPTEMBER 17TH,
2024 AND JANUARY 2 EIGHTH, 2025.
JUNE THIRD, 2025, AND SEPTEMBER
EIGHTH, 2025.
THESE ARE POSTED ON OUR WEBSITE.
THANKS FOR YOUR ATTENTION.
TURNING BACK TO DR. CHRISTY.
>> OKAY. I HOPE YOU HAVE SEEN
THE AGENDA. I WILL START BY
EXPRESSING GREAT APPRECIATION
FOR ELLEN WHO IS RETIRING FROM
COUNCIL AND RECOGNIZE WHAT A
TIME COMMITMENT THIS HAS
ENTAILED AND DEEPLY APPRECIATE
HER DEDICATION TO SERVICE.
OKAY. ALL RIGHT. THANK YOU TO
DR. GABO LEEZI AND WOULD LIKE TO
INTRODUCE A FEW NEW COUNCIL
MEMBERS, ONE PERMANENT AND 3 AD
HOC AND WILL INTRODUCE KARL
JEFFSON WHO THIS IS FIRST
MEETING OF FOUR-YEAR TERM AND
PLEASED TO REPORT WE HAVE 7
OTHER UPCOMING COUNCIL MEMBERS
THAT WE ARE EXPECTING TO BE
JOINING US IN SEPTEMBER.
WE ARE REALLY EXCITED TO WELCOME
ALL OF THEM AND DR. JEPSON IS
ASSOCIATE DEAN OF RESEARCH AND
PROFESSOR OF ORTHOPEAKEDIC
SURGERY AND BIOENGINEERING
PRIMARY FOCUS OF RESEARCH HAS
BEEN HOW COMPLEX ADAPTIVE
SYSTEMS LIKE BONE ESTABLISH
FUNCTION DURING GROWTH AND
MAINTAIN FUNCTION DURING AGING
AND RECENTLY LAB DISCOVERED
NOVEL ASSOCIATION BETWEEN
MEASURE OF EXTERNAL BONE SIZE
AND INTERNAL REMODELING PROCESS
THAT IS CENTRAL TO ESTABLISHING
AND MAINTAINING BONE STRENGTH.
DR. JEPSON IS A PROLIFIC
INVESTIGATOR AND PUBLICATIONS
AND NUMEROUS GRANTS AWARDS AND
DELIGHTED TO HAVE HIM JOINING
THE COUNCIL.
AS WE DID WITH OUR PAST FEW
MEETINGS, WE INVITED OUTSTANDING
INVESTIGATORS FROM NIAMS POOL OF
MENTORED KO1, KO8, AND K23 AWARD
RECIPIENTS TO PARTICIPATE IN THE
COUNCIL MEETING AS AD HOC
MEMBERS.
IDEA IS TO BOTH EXPOSE THEM TO
COUNCIL AND IMPORTANTLY FOR US
TO OBTAIN INPUT FROM SCIENTISTS
AT AN EARLIER CAREER STAGE THAN
COUNCIL MEMBERS TYPICALLY ARE.
TODAY, WE ARE JOINED BY TAMIA
HARRISON TRANAT UNIVERSITY OF
TEXAS SOUTHWESTERN MEDICAL
CENTER AND [INDISCERNIBLE] OF
TUFTS UNIVERSITY AND
[INDISCERNIBLE] OF JOHNS HOPKINS
UNIVERSITY. DR. TRAN -- SKIN
MICROBIOTA AND HOW THEY IMPACT
THE SKIN'S IMMUNE SYSTEM. DR.
CAST YOU ARY IS ASSISTANT
PROFESSOR OF MEDICINE DEVELOPING
AND TESTING MEASUREMENT OF
PATIENT CENTERED OUTCOMES IN
LUPUS AND INVESTIGATING
IMPLEMENTATION OF PATIENT
REPORTED OUTCOME MEASURES TO
IMPROVE ROUTINE CARE FOR
INDIVIDUALS WITH LUPUS. DR. MEG
ALLEY IS ASSISTANT PROFESSOR OF
MEDICINE AND INCLUDES
ASSOCIATION BETWEEN CANCER AND
MYOCITIS AND PATIENT REPORTED
OUTCOME MEASURES AND AUTO
ANTIBODIES AND REALLY
APPRECIATED GETTING THE
OPPORTUNITY TO SPEAK WITH CLIGS
TOEF FER LAST NIGHT AT THE
COUNCIL DINNER. PLEASURE AND
WARM WELCOME TO ALL OF YOU AND
FEEL FREE TO PARTICIPATE FULLY.
WE LOVE HEARING FROM YOU. YOU
HAVE VALUABLE PERSPECTIVES AND
REPORT TODAY WILL FOCUS IN THREE
GENERAL AREAS AND WILL REVIEW
STATUS OF NIH AND NIAMS BUDGETS
AND HIGHLIGHT PERSONAL CHANGES
WIDELY AT NIH AND AT NIAMS AND
WILL HIGHLIGHT ACTIVITIES OF
GENERAL INTEREST TO YOU.
SO, I WILL START BY PROVIDING
OVERVIEW AND UPDATE ON NIH AND
NIAMS BUDGET. YOU MAY KNOW BACK
ON MARCH 23RD, PRESIDENT BIDEN
SIGNED FURTHER CONSOLIDATED
APROPRIATION ACT OF 2024 INTO
LAW AND IT PROVIDED FUNDING TO
NIH FOR THE CURRENT FISCAL YEAR
THAT CONCLUDES IN SEPTEMBER 30TH
OF 2024.
SO, QUITE LATE IN THE YEAR FOR
US TO GET THAT FINAL -- THOSE
FINAL FIGURES.
THAT BILL PROVIDES 47.4 BILLION
DOLLARS FOR THE NIH THAT IS A
.7% DECREASE FROM THE LAST
FISCAL YEAR AND NIAMS RECEIVED
AN OPERATING BUDGET OF 687.6
MILLION THAT IS THE SAME AS LAST
YEAR.
WE HAVE A FLAT BUDGET.
WE ARE GRATEFUL TO CONGRESS,
HOWEVER, FOR CONTINUED SUPPORT
DURING CHALLENGING BUDGETARY
TIMES FOR RECOGNITION OF
IMPORTANT -- NOW, ON MARCH 11TH,
SO SHORTLY BEFORE THE PASSAGE OF
THE CURRENT FISCAL YEAR BUDGET,
PRESIDENT BIDEN ALSO RELEASED
FISCAL YEAR 2025 BUDGET REQUEST
TO CONGRESS THAT WOULD BEGIN
OCTOBER 1ST THIS YEAR AND AMOUNT
REQUESTED FOR NIH IS 51.6
BILLION WHICH IS INCREASE OF 5%
OVER LAST YEAR'S FINAL BUDGET.
FOR NIAMS, AMOUNT IN HIS BUDGET
IS 690 MILLION AND WOULD
REPRESENT INCREASE OF .3% OVER
LAST YEAR'S BUDGET.
THERE IS A LONG WAY TO GO BEFORE
WE UNDERSTAND WHAT BUDGETS WILL
BE FOR NEXT YEAR AND NEXT FEW
MONTHS HOUSE AND SENATE WILL
DEVELOP THEIR OWN BUDGET
PROPOSALS FOR FISCAL YEAR 2025
AND THOSE WILL BE RECONCILED AND
FINAL BILL WILL GO TO THE
PRESIDENT FOR HIS SIGNATURE.
NOW, BASED ON FINAL NUMBERS WE
GOT BACK IN MARCH FOR FISCAL
2024, OUR CURRENT YEAR, WE
UPDATED OUR FUNDING PLAN ON
NIAMS WEBSITE.
IT SHOWS A PAY LINE FOR MOST
INVESTIGATORS SET AT 8%, VERY
TIGHT PAY LINE.
I WANT TO EMPHASIZE AS WE HAVE
HAD SPOKEN ABOUT IN PREVIOUS
SESSIONS THAT THIS LOWER PAY
LINE SOMEWHAT LOWER IN LAST
COUPLE YEARS DOESN'T MEAN WE
WOULD PAY FEWER R01 GRANTS BUT
INMEANS WE ARE EMPOWERING
PROGRAM DIRECTORS TO
APPLICATIONS ABOVE THE PAY LINE
TO DECIDE WHICH ONES SHOULD BE
RECOMMENDED FOR FUNDING AND YOU
CAN SEE IN THE SLIDE, HOWEVER,
WE CONTINUE TO HAVE A GENEROUS
PAY LINE FOR R01 APPLICATIONS
COMING IN FROM EARLY STAGE
INVESTIGATARS AND THAT PAY LINE
IS 15% EARLY STAGE, SAME AS LAST
YEAR.
AS NOTED EARLIER AND
COMMUNICATED WIDELY WITH COUNCIL
MEETINGS NEWSLETTERS DIRECTORS
LETTERS PUBLISHED ON THE
WEBSITE, WE ARE MOVING FORWARD
AND BROADENING SUPPORT FOR
MISSION BY EXPANDING FUNDING
APPROACH AND WANT TO GO THROUGH
THIS AGAIN AND WE CAN DISCUSS IT
LATER IF THERE ARE ADDITIONAL
QUESTIONS.
HISTORICALLY NIAMS PAID MOST
APPLICATION AS CORDING TO A
STRICT PAY LINE DRIVEN APPROACH
AND LIMITED FUNDS AVAILABLE TO
AWARD APPLICATIONS IDENTIFIED AS
BEING HIGH PRIORITY THAT SCORED
BEYOND THE PUBLISHED PAY LINE
AND THOSE DECISIONS WERE MEANT
AT VERY END OF FISCAL YEAR AND
DOLLARS FLOWED BACK TO THE
INSTITUTE THAT WE HAD TO SPEND
AND STARTING THIS YEAR MOVING
FORWARD NIAMS WILL SET ASIDE
INCREASED FUNDING TO SUPPORT
APPLICATIONS DEEMED IMPORTANT TO
NIAMS PORTFOLIO SCORING BEYOND
THE PAYLINE AND REFER TO THE
PROCESS AS SELECT PAY.
EVOLVING APPROACH WILL INCREASE
FUNDS AVAILABLE FOR HIGH
PRIORITY PROJECTS ALLOWING
INSTITUTE TO BETTER ADDRESS GAPS
AND OPPORTUNITIES IN BROAD
PORTFOLIO MISSION AREAS AND
SUPPORT WIDE SPAN OF SCIENTIFIC
PERSPECTIVES AND INVESTIGATIVE
APPROACHES.
IMPORTANTLY, I WANT TO NOTE THAT
TOTAL AMOUNT OF FUNDS AVAILABLE
FOR NEW AWARDS MEANING THOSE
FUNDED ACCORDING TO PAY LINE OR
SELECT PAY REMAINS THE SAME AND
WE ARE NOT FUNDING IN REDUCED
NUMBER OF AWARDS BUT ARE
ALLOWING OR SETTING ASIDE MORE
OF OUR FUNDS THROUGHOUT THE YEAR
FOR OUR SELECT PAY PROCESS.
THAT ALSO HAS THE BENEFIT FOR
THOSE WHO -- THOSE APPLICATIONS
THAT ARE ELIGIBLE FOR SELECT PAY
FOR DECISIONS TO BE MADE EARLIER
IN THE YEAR INSTEAD OF WAITING
UNTIL END OF THE FISCAL YEAR
THAT PRESENTS A HARDSHIP FOR
INVESTIGATORS AND RECOGNIZE THAT
IN CURRENT BUDGET LANDSCAPE,
AMOUNT OF FUNDS AVAILABLE FOR
GRANTS MAY NOT KEEP UP WITH
INFLATION AND WITH REALITY WE
ARE COMMITTED TO MAINTAINING
STEADY NUMBER OF AWARDS EACH
YEAR TO HELP PRESERVE AND
STABILIZE RESEARCH WORKFORCE
THAT IS A GOAL SHARED ACROSS THE
NIH.
NOW I WILL MOVE ON TO PERSONNEL
CHANGES AT NIH AND NIAMS AND
AFTER A NATIONWIDE SEARCH MONICA
BER CONTINUE OELI NIH DIRECTOR
SELECTED SEAN MOONEY AS DIRECTOR
OF CENTER FOR INFORMATION
TECHNOLOGY AND I HAVE SEEN HIM
IN ACTION FOR A COUPLE MONTHS
NOW AND SERVE ON COMMITTEES
TOGETHER. HE IS FANTASTIC AND
HIT THE GROUND RACING AND GIVING
YOU MORE ABOUT HIS BACKGROUND HE
JOINED NIH IN MARCH A FEW MONTHS
AGO AND WAS PREVIOUSLY IN SOOE
AT SERVING AS PROFESSOR OF BIO
-- AND AS PART OF THAT ROLE, HE
SERVED AS THE CHIEF RESEARCH
INFORMATION OFFICER AND INTERIM
DIRECTOR FOR UNIVERSITY OF
WASHINGTON INSTITUTE FOR MEDICAL
DATA SCIENCE AND WAS DIRECTOR
FOR INFORMATICS FOR UNIVERSITY
OF WASHINGTON INSTITUTE OF
TRANSLATIONAL SCIENCES AND HAD
LOTS OF ROLES AND PREVIOUS
EARLIER TRAINING WAS IN
CHEMISTRY AND INFORMATICS AND
RESEARCH INTEREST FOCUSED ON
LEVERAGING COMPUTATIONAL CYBER
INFRASTRUCTURE IN DATA SCIENCE
AVEND IS A FELLOW OF AMERICAN
COLLEGE OF MEDICAL INFORMATICS
EARNING BACHELORS IN
BIOCHEMISTRY AND MOLECULAR
BIOLOGY FROM THE UNIVERSITY OF
WISCONSIN AND RECEIVED PHD IN
PHARMACEUTICAL CHEMISTRY FROM
USF AND AMERICAN CANCER SOCIETY
JOHN HOPKINS FELLOW AT STANFORD
IN DEPARTMENT OF GENETICS AND
MEDICAL INFORMATICS AND HIGHLY
QUALIFIED AND WILL BE A
FANTASTIC ADDITION TO THE
COMMUNITY.
NIH ALSO WELCOMES DR. KATHLEEN
NOOUZAL AS DIRECTOR OF FOGARTY
INTERNATIONAL CENTER AND IN ROLE
SERVES AS NIH ASSOCIATE DIRECTOR
FOR NIH RESEARCH AND IS HIGHLY
QUALIFIED AND FIRST WOMAN TO
HOLD PERMANENT DIRECTORSHIPS
SINCE CENTER'S FOUNDING BACK IN
1968 AND PRIOR TO JOINING NIH
WAS ENDOWED PROFESSOR IN
VAXINOLOGY AND DIRECTOR FOR
CENTER FOR VACCINE DEVELOPMENT
AND GLOBAL HEALTH AND WAS CHIEF
OF DIVISION OF GEOGRAPHIC
MEDICINE AT UNIVERSITY OF
MARYLAND SCHOOL OF MEDICINE IN
BALTIMORE AND RECEIVES
UNDERGRADUATE DEGREE AT
UNIVERSITY OF MARYLAND AT
COLLEGE PARK AND SHE COMPLETED
INTERNSHIP AND RESIDENCY AT
VANDERBILT AND COMPLETED
FELLOWSHIP IN INFECTIOUS
DISEASES AND EARNED MASTERS OF
PUBLIC HEALTH AND MEMBER OF
NATIONAL ACADEMY OF MEDICINE AND
SHE PREVIOUSLY SERVED ON CDC'S
ADVISORY COMMITTEE ON
IMMUNIZATION PRACTICES.
ALSO HIGHLY QUALIFIED FOR THIS
VERY IMPORTANT ROLE.
NOW, WHERE ARE THE TISSUES?
HERE AT NIAMS BOB CARTER
ANNOUNCED INTENTION TO RETIRE
FROM GOVERNMENT SERVICE AT END
OF CALENDAR YEAR.
SO FOR THOSE WHO HAVE NOT BEEN
PART OF OUR COMMUNITY, BEFORE
BOB JOINED, BOB JOINED NIAMS
2008 AND STEPPED IN AS ACTING
DIRECTOR FOLLOWING PASSING IN
2018 AND JOINED INSTITUTE IN
2021 AND BOB HAS MADE SURE THAT
NIAMS INTEREST HAS BEEN
PROMINENTLY FEATURED IN SEVERAL
TRANS NIH PARTNERSHIPS AND
PRESENCE IN WELL-BEYOND WHAT
WOULD BE EXPECTED BASED ON
RELATIVELY SMALL SIZE OF THE
INSTITUTE AND EVENT MAJOR
IMPORTANT PLAYERS AND LEADERS IN
ACCELERATING MEDICINES
PARTNERSHIP PROGRAM AND BACK
PAIN CONSORT SHUIUM AND BACK
PATH AND REDUCE PAIN OR REJOINED
CONSORTIUM AND BOTH PROGRAMS ARE
PART OF NIH'S HEEL INITIATIVES
AND ALLOWS TO LEVERAGE RESOURCES
DON'T COME OUT OF OUR BUDGET AND
IS IMPORTANT AND JOINING
INSTITUTE AS A TOTAL NEW BY TO
THE NIH AND CONSISTENT
LEADERSHIP ON ALL THESE FRONTS.
WE WILL BE SORRY TO SEE HIM GO
AND COMMITTED TO CONTINUE IN
CERTAIN AREAS FOLLOWING
RETIREMENT AND THANKS FOR YOUR
SERVICE, BOB.
YOU WILL SPEAK TO THE GROUP
LATER TODAY BUT IF YOU WANT TO
SHARE COMMENTS.
MOVING ON SOME REALLY GREAT NEWS
NOW.
FIRST OF ALL, MOST KNOW DR.
[INDISCERNIBLE] ANNOUNCED
INTENTION TO STEP DOWN AS
SCIENTIFIC DIRECTOR LAST YEAR
AND CONTINUED IN REMAINING
POSITION CONDUCTING NATIONWIDE
SEARCH FOR THIS IMPORTANT
LEADERSHIP ROLE AND REMAIN
APPRECIATIVE OF JOHN'S ONGOING
LEADERSHIP AND KNOW HE LOOKS
FORWARD TO SPENDING TIME
PROMOTING DIVERSITY AMONG NIAMS
WORKFORCE AND COMMITTED MENTOR
MAKING DISCOVERIES IN VERY BUSY
PRODUCTIVE LABS IN HIS GREAT
PASSIONS AND MENTIONING DURING
LAST ADVISORY COUNCIL WE
RECEIVED A ROBUST RESPONSE TO
POSITION ANNOUNCEMENT AND
ALTHOUGH WE WILL CONTINUE TO
ACCEPT APPLICATIONS UNTIL
POSITION IS FILLED WE IDENTIFIED
SEVERAL OUTSTANDING CANDIDATES
AND NOW INVITED TOP CANDIDATES
TO COME AND VISIT NIAMS AND GIVE
PRESENTATIONS AND MEET WITH
PROGRAM MEMBERS AND THESE VISITS
BEGIN IN MID JUNE AND GO ABOUT
MID-JULY. I WILL KEEP YOU
INFORMED ABOUT THE PROGRESS AND
WE ARE REALLY EXCITED HOW THE
PROCESS IS GOING.
SO MOVING ON TO EXTRAMURAL
RESEARCH PROGRAMS, I'M EXCITED
TO INTRODUCE TWO NEW PROGRAM
DIRECTORS JESS EPEE WHO JOINED
FROM LDI AND SERVED AS CHAIR OF
-- OVER 10 YEARS.
INTERIM NHLBI TRAINING DIRECTOR
OVER 2 YEARS PRIOR TO JOINING
NIH BACK IN 2010, DR. FIDUCHI OR
WHEN HE JOINED NIH BACK IN 2020,
INITIAL ROLE WAS AS A SCIENTIFIC
REVIEW OFFICER WITHIN CLINICAL
STUDIES AND TRAINING BRANCH IN
LBI AND SPANNING -- PARTICULAR
FOCUS ON THE K99RO GRANT
APPLICATIONS.
OBTAINED PHD IN BIOLOGY AND
MEDICAL BIOCHEMISTRY FOR
UNIVERSITIES IN ITALY AND DURING
POST DOC STUDIES ISOFORMS OF
EXTRA CELLULAR LABORATORIES AT
RIF KIN NEW YORK SCHOOL OF
MEDICINE AND PROFESSOR AT SAME
INSTITUTION INVESTIGATED COMPLEX
INTERPLAY -- INFLAMMATORY PROET
TEE AZS AND NIH FUNDED WORK
HIGHLIGHTS ROLE OF THROMBBANT
AND MITOGIN ACTIVATED KINASES
AND RESULTED IN NUMEROUS PEER
REVIEW PUBLICATIONS AND PATENT
AND AT NIAMS DR. PENN TUCHI WILL
HELP WITH THE ORTHOPEDICS
PORTFOLIO AND HIGHLY QUALIFIED.
ALSO, I'M DELIGHTED TO INTRODUCE
ISA VINCENT JOINING FROM NIGMS
AND HE SERVED AS SCIENTIFIC
REVIEW OFFICER IN CHARGE OF
STANDING REVIEW PANEL TWDA WHICH
REVIEWED A LION'S SHARE OF
PREDOCTORAL T32 APPOINTMENTS
LABS AWARDED BY NIH AND BEGAN
SERVICE AS HEALTH SCIENTIST AS
OFFICE OF DISEASE PREVENTION AND
PRIOR TO JOINING NIH WAS AT
UNIVERSITY OF VIRGINIA AND POST
DOCTORAL RESEARCH FOCUSED ON
FIBROSIS AND REPAIR AND
SIGNALING IN LAB OF DR. MARK
OPEWSA AND DR. VINCENT WILL
OVERSEE AND COORDINATE NIAMS
TRAINING AND PORTFOLIO WILL
INCLUDE T32S AND F AWARDS AND
K99 PORTION OF THE PARENT AND
MOSAIC K99R0 AWARDS IN ADDITION
TO COORDINATING ADMINISTRATION
OF K AWARDS AND HAS BEEN
FABULOUS INTERACTING WITH BOTH
OF THESE PEOPLE AND REALLY
EXCITED TO HAVE YOU ON BOARD.
WE ARE ALSO WELCOMING DR. SHRONG
FANG AS NEW SCIENTIFIC DIRECTOR
AND HE STUDIED REGULATION OF
IMMUNE RESPONSE IN ALLERGIC LUNG
IN MOUSE MODELS AND BEFORE
JOINING NIEHS WORKED AS A
RESEARCH ASSOCIUSASE AND LATER
PROFESSOR AT UNIVERSITY OF
MARYLAND SCHOOL OF MEDICINE AND
FOCUSED ON ROLE OF -- HOST
PATHOGEN INTERACTIONS AND
MUCOSAL IMMUNE RESPONSES AS WELL
AS VACCINE DEVELOPMENT.
WELCOME TO ALL OF YOU.
NEXT I WOULD LIKE TO HIGHLIGHT
NIH AND NIAMS ACTIVITIES MIGHT
BE OF INTEREST TO YOU AND
BROADER COMMUNITY AND MARCH 18TH
I WAS HONORED TO JOIN NIH
DIRECTOR MONICA BERTAGNOLLI AND
NIH LEADERS AT WHITE HOUSE AND
PRESIDENT JOE BIDEN SIGNED
EXECUTIVE ORDER ANNOUNCING
HEALTH RESEARCH AND INNOVATION
AND ORDER ASPIRES TO INTEGRATE
WOMEN'S HEALTH ACROSS FEDERAL
RESEARCH PORTFOLIO AND
PRIORITIZE INVESTMENTS IN
WOMEN'S HEALTH RESEARCH
GALVANIZES RESEARCH ON WOMEN'S
MIDLIFE HEALTH AND HELP ASSESS
UNMET NEEDS TO SUPPORT WOMEN'S
HEALTH RESEARCH.
SO WOMEN'S HEALTH IS AN ISSUE OF
GREAT CONGRESSIONAL INTEREST AND
ON MAY SECOND, SENATORS LISA AND
PATTY MURRAY AND TAMMY BALDWIN
SUSAN COLLINS AMY CLOEB ASSURE
INTRODUCED BIPARTISAN ADVANCING
MENOPAUSE CARE AND MIDLIFE
WOMEN'S HEALTH ACT TO BOOST
RESEARCH ON MENOPAUSE AND
COORDINATE FEDERAL GOVERNMENT'S
EXISTING PROGRAMS RELATED TO
MIDLIFE WOMEN'S HEALTH BILL THAT
IS AUTHORIZED FOR 5 YEARS AIMS
TO STRENGTHEN AND EXPAND FEDERAL
RESEARCH ON MENOPAUSE HEALTH
CARE WORKFORCE TRAINING
AWARENESS EDUCATION EFFORTS AND
PUBLIC HEALTH PROMOTION AND
PREVENTION TO ADDRESS MENOPAUSE
AND WOMEN'S MIDLIFE HEALTH
ISSUES.
RESEARCH ON WOMEN'S HEALTH HAS
ALWAYS BEEN A FOCUS ON THE NIH.
TWO WEEKS AGO NIH DIRECTOR
MONICA SHARED HER VISION FOR
FUTURE OF RESEARCH ON WOMEN'S
HEALTH AND LAUNCHED NIH-WIDE
STRATEGIC PLAN FOR RESEARCH ON
HEALTH OF WOMEN 2024 TO 2028 AND
PLAN SERVES AS A ROAD MAP TO
GUIDE AND INFORM NIH SUPPORTED
RESEARCH ON DISEASES AND HEALTH
CONDITIONS THAT UNIQUELY EFFECT
WOMEN AND NIH AND OFFICE OF
RESEARCH AND WOMEN'S HEALTH
EXTEND BEYOND THE LABORATORY
ACTIVELY SEEKING TO EMPOWER
WOMEN AND GIRLS ADDRESSING SEX
AND GENDER DISPARITIES AND
HEALTH OUTCOMES PROMOTING
RESEARCH INCLUDING WOMEN ACROSS
THE LIFE FORCE AND SUPPORTING
WOMEN IN BIOMEDICAL CAREERS.
NOW SWITCHING TO CONGRESSIONAL
INTERACTION I WAS INVITED TO
PARTICIPATE IN A BRIEFING RELATE
THE TO SKIN DISEASE RESEARCH
WITH CONGRESSIONAL MEMBERS AND
STAFF AS PART OF THE FIELD DAY
ACTIVITIES ORGANIZED BY
COALITION OF SKIN DISEASES IF I
RECALL CORRECTLY THEY HAD
SOMETHING LIKE 500 MEETINGS
SCHEDULED WITH VARIOUS STAFF AND
WAS IMPRESSIVE AND WE ARE
GRATEFUL TO HAVE OPPORTUNITIES
LIKE THIS TO HIGHLIGHT
OUTSTANDING WORK WE SUPPORT AND
EDUCATE CONGRESS ABOUT NIAMS MAY
IS LUPUS AWARENESS MONTH AND
COMMUNITY ACTIVE IN RAISING
AWARENESS ABOUT THIS CHRONIC
DISEASE AND DURING 2024 NATIONAL
LUPUS ADVOCACY SUMMIT ORGANIZED
BY LUPUS FOUNDATION SUMMIT IN
AMERICA -- UNDERWAY WITHIN THE
FEDERAL GOVERNMENT ON LUPUS
RESEARCH AND EDUCATION AND THIS
IS THE FIRST TIME I ATTENDED
THIS EVENT AND WAS IMPRESSED AS
A PARTICIPATE IN THESE EVENTS
AND MEETINGS WE HAD WITH
ORGANIZATIONS INTERESTED IN
RESEARCH RELATED TO BONES JOINTS
MUSCLES AND SKIN I'M IMPRESSED
WITH HOW MUCH MISSION IS DRIVEN
WITH MEANINGFUL INTERACTIONS
WITH PATIENT ADVOCACY AND
COMMUNITY GROUPS AND PATIENTS
AND CAREGIVERS ARE AT HEART OF
WHY WE DO WHAT WE DO AND
ENGAGEMENT WITH PATIENT ADVOCACY
AND COMMUNITY GROUPS IS CORE TO
MISSION AND NIAMS DIRECTOR
COMMITTED TO ENSURING VOICES ARE
ACKNOWLEDGED AND VITALLY
IMPORTANT FEEDBACK IS
INCORPORATED INTO INSTITUTE'S
ACTIVITIES AND DURING TODAY'S
MEETING AND NEXT FEW COUNCIL
MEETINGS WE WILL FOCUS ON
PATIENT AND COMMUNITY ENGAGEMENT
THAT IS CRITICALLY IMPORTANT AND
TIMELY AND INVITED PATIENT
REPRESENTATIVES FROM NIAMS
MISSIONARIES TO SPEAK AT TODAY'S
MEETING AND LOOK FORWARD TO
HEARING FROM THEM.
I WILL NOW SHARE UPDATES ON
PROGRAMS AND INITIATIVES THAT
ARE OF INTEREST TO NIAMS AND
PREVIOUS COUNCIL MEETINGS I TOLD
YOU ABOUT EXACT THAT STANDS FOR
EXPOSOME AND AUTO IMMUNE DISEASE
COORDINATING COLLABORATING TEAMS
AND TURNED OUT TO BE INAUGURAL
PROGRAM OF OFFICE OF AUTO IMMUNE
DISEASE RESEARCH AND IN MARCH
NIH MADE 6 AWARDS UNDER PROGRAMS
FIRST STAGE OF INITIATIVE THAT
IS CALLED EXACT PLAN WITH PLAN
STANDING FOR PLANNING AWARD R21
PLANNING AWARDS AND DISEASE
FOCUS OF SOME AWARDS INCLUDE
TOPICS WITHIN NIAMS MISSION SUCH
AS HUMETOID ARTHRITIS LUPUS AND
SORATIC DISEASE AND HAD FIRST
ANNUAL SUMMIT TO TALK ABOUT
OPPORTUNITIES AND CHALLENGES IN
THIS AREA EARLIER THIS MONTH.
NOW, AS MANY OF YOU MAY RECALL
NITRO WHICH STANDS FOR NOVEL
INNOVATIONS TISSUE REGENERATION
AND OSTEOARTHRITIS WAS FIRST
ARPA H PROGRAM TO BE LAUNCHED
AND EXPRESSING THANKS TO NIAM
STAFF THAT PROVIDED SUBSTANTIAL
TECHNICAL ASSISTANCE TO ARPA H
AS THEY DEVELOPED THIS PROGRAM
AND PROGRAM SUBSEQUENTLY
SELECTED 5 NITRO PERFORMER TEAMS
DEVELOPING AND LEVERAGING
INNOVATIVE FORMS OF REGENERATIVE
MEDICINE AND THERAPEUTICS THAT
FULLY REPAIR DAMAGED JOINTS AND
EXCITING GOALS AND THESE
PERFORMER TEAMS INCLUDE A TEAM
LED BY DUKE UNIVERSITY FOCUSING
ON TIME RELEASE OF THERAPIES AND
TEAM LED AT WASHINGTON
UNIVERSITY AT ST. LOUIS FOCUSING
ON SMART CELL THERAPY AND TEAM
AT UNIVERSITY OF COLORADO
BOULDER THAT FOCUSES ON
ENGINEERED MATERIALS AND LED BY
FOCUSING ON INTEGRATED TOTAL
KNEE IMPLANTS AND TEAM BY CASE
WESTERN UNIVERSITY FOCUSING ON
MODULAR TOTAL KNEE IMPLANTS AND
PROVIDE TECHNICAL ASSISTANCE TO
NEW PROGRAM AS NEEDED AND
LOOKING FORWARD TO ACHIEVEMENTS
AND MANY THANKS TO OTHERS
STAYING ENGAGED WITH THIS
INITIATIVE.
NIH RECOGNIZES DRAMATIC LEAPS
AND TECHNOLOGIES THAT ALLOW
RESEARCHERS TO USE COMPLIMENTARY
AND THESE NEW APPROACH
METHODOLOGIES THAT ARE BEING
CALLED NAMS THAT ARE INCLUDING
COMPUTATIONAL MODELING AND
PREDICTIVE TECHNOLOGIES CELL
ASSAYS AND CELL BASED CULTURE
METHODS AND CELL BASED CULTURE
MODELS HOLD TREMENDOUS WHEN
APPLIED TO SCIENTIFIC INQUIRY
AND COMPLEMENT OF ANIMAL
RESEARCH AND EXPERIMENTATION IS
NEW NIH COMMON FUND PROGRAM
AIMING TOAC CELL RATE
DEVELOPMENT AND STANDARDIZATION
AND VALIDATION AND USE OF NEW
METHODS AND APPROACHES THAT WILL
ACCURATELY MODEL HUMAN BIOLOGY
AND COMPLEMENT OR IN CASES
REPLACE TRADITIONAL MODELS
MAY 7TH, COMPLEMENTARY ANNOUNCED
WINNERS OF CROWDSOURCING
COMPETITION FOR INNOVATIVE IDEAS
ON NAMS, THESE NEW APPROACH
METHODOLOGIES AND EACH OF THE 20
WINNING TEAMS WON $50,000 FOR
INNOVATIVE SOLUTIONS AND JUDGING
CRITERIA DEMONSTRATING OUT OF
THE BOX SOLUTIONS ACROSS
COMPETITION AREAS OF INSILICO,
IN VITRO, INCHEMICO AND COMBIN
TORIAL METHODS AND WINNING
SOLUTIONS OF INTEREST TO NIAMS
INCLUDE IDEAS TO DEVELOP A
NEUROIMMUNOCUTANEOUS HUMAN SKILL
EQUIVALENT AND ORGAN ON CHIP FOR
STUDYING OSTEOARTHRITIS PAIN AND
ORGANOID MODELING OF RHEUMATOID
ARTHRITIS JOINTS AND IS GREAT
NEWS THAT SOME WINNERS ARE
FOCUSING ON TOPICS OF GREAT
INTEREST TO US.
SO MOVING ON TO SOME RESEARCH
RESULTS FROM THE MOLECULAR
TRANSDUCERS OF PHYSICAL ACTIVITY
CONSORTIUM OR MOTOR PACK, MOTOR
PACK IS AN NIH COMMON FUND
PROGRAM LED BY NIAMS NATIONAL
INSTITUTE OF AGING AND NIDDK AND
IS A NATIONAL RESEARCH
CONSORTIUM FIRST FUNDED IN 2017
TO DISCOVER AND PRELIM NARLY
CHARACTERIZE RANGE OF
TRANSDUCERS PHYSICAL ACTIVITY IN
HUMANS AND RATS OF VARIOUS AGES
AND PROGRAM'S GOAL IS TO STUDY
MOLECULAR CHANGES OCCURRING
DURING EXERCISE AND ADVANCE
UNDERSTANDING HOW PHYSICAL
ACTIVITY IMPROVES AND PRESERVES
HEALTH AND RESULTS FROM YOUNG
ADULT RATS THAT UNDERWENT A
TRAINING PROGRAM HAVE NOW BEEN
PUBLISHED AND RESEARCHERS FOUND
THAT BODILY TISSUES TESTED
RESPOND TO EXERCISE TRAINING AND
AMOUNTING TO OVER 35,000
BIOLOGICAL MOLECULES RESPONDING
AND ADAPTING TO ENDURANCE
EXERCISE OVER TIME INCLUDING
TISSUES FROM ORGANS NOT USUALLY
ASSOCIATED WITH INFECTIOUS
EXERCISE AND MOLECULAR CHANGES
SEEN IN ALL TISSUES WAY IN WHICH
EACH TISSUE RESPONDED WAS UNIQUE
AND THEY ALSO FOUND DIFFERENCES
IN RESPONSES BETWEEN MALE AND
FEMALE RATS THAT WERE MORE
WIDESPREAD THAN ANTICIPATED
HIGHLIGHTING IMPORTANCE OF
INCLUDING ANIMALS IN SEXES AND
PRECLINICAL RESEARCH AND DATA
PRODUCED THROUGH THIS RESEARCH
PROJECT IS PUBLICLY AVAILABLE ON
MOTOR PACK DATA HUB FOR FURTHER
ANALYSIS AND DIRECT DOWNLOAD TO
ENCOURAGE HYPOTHESES FROM
BIOMEDICAL COMMUNITY AND INVITE
MOTOR PACK INVESTIGATOR TO
PRESENT FINDINGS AT FUTURE
MEETING THAT IS EXCITING AND
UNIQUE PROGRAM AND BELIEVE IT
HAS BEEN A NUMBER OF YEARS
CERTAINLY BEFORE MY TIME SINCE
YOU HEARD ABOUT THIS PROGRAM AND
LOOKING FORWARD TO THAT
OPPORTUNITY.
SO, WITH ALL OF THE EXCITING
OPPORTUNITIES FOR NEW METH
OTDOLOGIES AND INVALUABLE DATA
FOR INTERROGATIONS SUCH AS MOTOR
PACK DATA SET I WOULD LIKE TO
SHARE A RESEARCH FROM NIH CENTER
FROM INFORMATION TECHNOLOGY AND
NIH OFFICE OF DATA SCIENCE
SHARING THAT ENABLES RESEARCHERS
TO TRY THE CLOUD IN A SECURE
NIH-APPROVED ENVIRONMENT.
NIH CLOUD LAB IS A NO-COST 90
DAY CLOUD ADOPTION PROGRAM
PROVIDING AN AMOUNT -- AN
ACCOUNT WITH 5 HUNDRED DOLLARS
OF AMAZON WEB SERVICE SERVICES
GOOGLE CLOUD OR MICROSOFT AZUR
CREDITS ACCESS TO CURATED
BIOINFORMATICS TUT TORE &S AND
SUPPORTS FROM NIH EXPERTS CLOUD
LAB AVAILABLE TO INTRAMURAL
RESEARCHERS AND NIH STAFF SINCE
JUNE 2022 AND NEWLY AVAILABLE TO
RECIPIENTS OF NIH FUNDING AND
RESEARCHERS INSTITUTIONS
ELIGIBLE FOR NIH FUNDING WHO
MIGHT NOT HAVE AN ACTIVE AWARD
AND PARTICIPANTS CAN SIGN UP ANY
TIME AND USE CLOUD LAB TO TRY
BEFORE YOU BUY AND EVALUATE IF
CLOUD IS A GOOD FIT FOR YOUR
PROJECT WITHOUT MAKING A
LONG-TERM COMMITMENT TO LEARN
NEW SKILLS ACCESSING TUTORIALS
TO DEMONSTRATE HOW TO RUN
REALISTIC BIOFORMATIC DATA
SCIENCE AND AI WORKFLOWS AND
DEVELOP NEW TOOLS PROTOTYPING
ARCHITECTURES AND EVALUATING
SOFTWARE AND HARDWARE
COMBINATIONS EXPLORING POTENTIAL
OF GENERATIVE AI AND PROGRAM
OFFICE OPEN TO RESEARCHERS WITH
ALL LEVELS OF CLOUD KNOWLEDGE
FROM VERY BEGINNERS TO
EXPERIENCE TEAMS AND IF YOU
WOULD LIKE TO LEARN MORE ABOUT
THIS OPPORTUNITY THAT IS NOW
AVAILABLE TO THE BROADER
COMMUNITY, YOU CAN SCAN THAT
Q.R. CODE OR WRITE TO US.
WE WILL SEND YOU TO THE RIGHT
PLACE.
SO, NEXT, I WOULD LIKE TO DRAW
YOUR ATTENTION TO AN EXCITING
NEW OPPORTUNITY.
THIS RECOGNIZES THE CRITICAL
NEED TO EXTEND RESEARCH
PARTICIPATION OPPORTUNITIES TO
BROADER COMMUNITIES.
ONE OF DR. BERTAGNOLLI'S NIH
DIRECTORS TOP PRIORITIES IS
BETTER SERVING RURAL COMMUNITIES
COMING FROM A VERY RURAL
ENVIRONMENT 100 MILES AWAY FROM
A CLIP IN WYOMING WHO IS
UNDERSTANDING OF CHALLENGES OF
COMMUNITIES AND APRIL FIFTH NIH
COUNCIL OF COUNCILS APPROVED
CONCEPT OF ESTABLISHING NETWORK
FOR CLINICAL RESEARCH AND
PRIMARY CARE THAT DR.
BERTAGNOLLI PRESENTED TO THEM
AND PROGRAM RECENTLY ANNOUNCED
PILOT RESEARCH OPPORTUNITY
CALLED INTEGRATING CLINICAL
RESEARCH INTO PRIMARY CARE
SETTINGS THROUGH NETWORK
RESEARCH HUBS AND PURPOSE OF THE
RESEARCH OPPORTUNITY
ANNOUNCEMENT IS INVITING
APPLICATIONS BY EXISTING
CLINICAL NETWORK RESEARCH AND
ORGANIZATIONS CURRENTLY
AFFILIATED AND PARTICIPATING IN
SPECIFIC EXISTING CLINICAL
RESEARCH NETWORKS SERVING AS
NETWORK RESEARCH HUB AND LARGER
NET INFRASTRUCTURE SUPPORTING
RESEARCH AND CARE SETTINGS AND
EFFORT WILL START SMALL AND
FOCUSED DR. BERTAGNOLLI HAS
GRAND PLANS HOW THIS COULD
EVOLVE OVER TIME.
OPPORTUNITY IS LIMITED TO
ORGANIZATIONS CURRENTLY THAT
FOCUS ON SERVING RURAL
COMMUNITIES AND COMPLETED
APPLICATIONS ARE JUNE -- THEY
ARE DUE JUNE 14TH.
SO, IN THE NEXT FEW MINUTES, I
WANT TO HIGHLIGHT SEVERAL
IMPORTANT NIH AND NIAMS POLICY
CHANGES AND AS DISCUSSED IN PAST
MEETINGS, IN FACT, YOU -- SOME
HEARD FROM DR. NONI BURN
DIRECTOR OF CENTER FOR
SCIENTIFIC REVIEW A FEW MEETINGS
BACK.
NIH IS IMPLEMENTING A SIMPLIFIED
FRAMEWORK FOR PEER REVIEW OF
MAJORITY OF COMPETING RESEARCH
PROJECT GRANT APPLICATIONS
BEGINNING WITH SUBMISSIONS WITH
DUE DATES JANUARY 2025.
SIMPLIFIED PEER REVIEW FRAMEWORK
AIMS TO BETTER FACILITATE
MISSION OF SCIENTIFIC PEER
REVIEW THAT IS IDENTIFYING
STRONGEST HIGHEST IMPACT
RESEARCH BY ENABLING PEER
REVIEWERS TO BETTER FOCUS ON
ANSWERING KEY QUESTIONS
NECESSARY TO ASSESS SCIENTIFIC
AND TECHNICAL MERIT OF PROPOSED
RESEARCH AND SHOULD PROPOSED
RESEARCH BE CONDUCTED AND CAN
PROPOSED RESEARCH PROJECT BE
CONDUCTED? WHILE ALSO
MITIGATING THE EFFECT OF
REPUTATIONAL BIAS BY FOCUSING
EVALUATION OF INVESTIGATOR AND
ENVIRONMENT TO WITHIN THE
CONTEXT OF THE PROPOSED RESEARCH
AND INTENDED TO REDUCE REVIEWER
BURDEN SHIFTING POLICY
COMPLIANCE ACTIVITIES TO NIH
STAFF AND SIMPLIFIED FRAMEWORK
FOR NIH PEER REVIEWED CRITERIA
RETAINS 5 REGULATORY CRITERIA
APPROACH ENVIRON PT AND
REORGANIZES THEM INTO THREE
FACTORS, 2 OF WHICH WILL RECEIVE
A NUMERICAL CRITERION SCORE AND
ONE WILL BE EVALUATED FOR BY
SUFFICIENCY AND ALL THREE
FACTORS WILL BE CONSIDERED IN
ARRIVING IN OVERALL IMPACT
SCORES AND I THINK I APPLAUD
GOALS OF REVISED REVIEW
CRITERION AND EAGER TO SEE THEM
IMPLEMENTED.
SO, CHANGES ARE ALSO COMING FOR
NIH FELLOWSHIP APPLICATION AND
REVIEW PROCESS AND MOTIVATED BY
SIMILAR CONCERNS OR GOALS AND
FELLOWSHIP APPLICATIONS SUBMIT
ON OR AFTER JANUARY 25TH 2025
WILL FOLLOW REVISED APPLICATION
AND REVISED REVIEW CRITERIA.
GOAL OF THE CHANGES IS TO
IMPROVE CHANCES THAT PROMISING
FELLOWSHIP CANDIDATES WILL BE
CONSISTENTLY IDENTIFIED BY
SCIENTIFIC REVIEW PANELS AND
CHANGES WILL BETTER FOCUS
REVIEWER ATTENTION ON THREE KEY
ASSESSMENTS.
FELLOWSHIP CANDIDATES
PREPAREDNESS AND POTENTIAL AND
RESEARCH TRAINING PLAN, AND
COMMITMENT TO THE CANDIDATE.
THE CHANGES WILL ALSO ENSURE
BROAD RANGE OF CANDIDATES AND
RESEARCH TRAINING CANDIDATES CAN
BE RECOGNIZED AS MERITORIOUS
CLARIFYING AND SIMPLIFYING
LANGUAGE AND APPLICATION OF THE
REVIEW CRITERIA AND ALSO CHANGES
WILL REDUCE BIAS AND REVIEW
EMPHASIZING COMMITMENT TO
CANDIDATE WITHOUT
UNDERCONSIDERATION OF SPONSOR
AND INSTITUTIONAL REPUTATION.
SO I ENCOURAGE ALL OF YOU TO
LEARN MORE ABOUT THE CHANGES AND
ADDITIONAL GUIDANCE RELATED TO
THAT ON NIH WEBSITE.
ANOTHER CHANGE THAT SOME OF YOU
MAY HAVE OR MAY ALREADY BE AWARE
OF, THIS IS RELATED TO THE
RECENT EVALUATION OF THE
IMMUNOLOGY AND RESPIRATORY
SYSTEMS CLUSTER OF STUDY SECTION
BUSINESS NIH CENTER FOR
SCIENTIFIC REVIEW.
CSR'S EVALUATING PANEL QUALITY
OR REVIEW OR INQUIRE PROGRAM
INTEGRATES DATA AND INPUT FROM
MULTIPLE STAKEHOLDERS TO
DETERMINE IF CHANGES IN STUDY
SECTION FOCUS OR SCOPE ARE NEED
TODAY FACILITATE IDENTIFICATION
OF HIGHLY IMPACTED SCIENCE WITH
SPERNL CONSIDERATION OF EMERGING
AREAS OF SCIENCE.
EVALUATION OF IMMUNOLOGY AND
RESPIRATORY SYSTEM CLUSTER
INCLUDING ARTHRITIS CONNECTIVE
TISSUE OR SKIN OR ATS SECTION
THAT MANY ARE FAMILIAR WITH
STARTED LAST YEAR AND RESULT OF
THAT EVALUATION, MOST
APPLICATIONS THAT WERE
PREVIOUSLY REVIEWED BY ACTS WILL
NOW BE REVIEWED BY NEW STUDY
SECTION CALLED SKIN AND
CONNECTIVE TISSUE SCIENCES OR
SCTS.
WITH REMAINING TO BE REVIEWED BY
MECHANISMS OF AUTO IMMUNITY OR
MAI STUDY SECTION AND SMALL
NUMBER OF APPLICATIONS TO BE
REFER TODAY IMMUNE MECHANISMS OF
HYPERSENSITIVITY ORAL EARTH JI
OR IMHA STUDY SECTION AND
IMPLEMENTATION OF CHANGES IS
ONGOING BETWEEN NOW AND
APPROXIMATELY THIS FALL AND THIS
INQUIRE PROGRAM IS ONGOING
ASSESSMENT PLAN ACROSS ALL STUDY
SECTIONS TO ENSURE THAT WE
CONTINUE TO THINK ABOUT FOCUS
AND SCOPE AND MAKE SURE THAT
STUDY SECTIONS KEEP UP WITH
SCIENCE.
WHEN WE MET LAST TIME, I SHARED
WITH YOU THE VERY STRONG
RECOMMENDATIONS FROM NIH
ADVISORY COMMITTEE TO THE
DIRECTOR WORKING GROUP
REENVISIONING NIH SUPPORTED POST
DOCTORAL TRAINING WORKING GROUP
CHARGED BY NIH DIRECTOR TO
REENVISION POST DOCK TORIAL
EXPERIENCE SO IT IS SUSTAINABLE
AND INCLUSIVE AND BETTER
RECOGNIZE AND REWARD VALUE OF
POST DOCTORAL SCHOLARS THIS US
SCIENTIFIC ENTERPRISE AND
WORKING GROUP'S TOP
RECOMMENDATION WAS FOR NIH TO
INCREASE PAY AND BENEFITS FOR
NIH SUPPORTED POST DOCTORAL
SCHOLARS AND THAT PROCESS HAS
OCCURRED INTERNALLY WITHIN
INTRAMURAL RESEARCH PROGRAM HERE
AND IN APRIL NIH ANNOUNCED
INCREASING ANNUAL PAY LEVELS FOR
PREDOCTORAL AND DOCTORAL
SCHOLARS AT NIH FUNDED EXTERNAL
INSTITUTIONS RECIPIENTS OF RUTH
L KOOERSTEIN NSA AWARDS AND
PREDOCTORAL SCHOLARS WILL
INITIALLY RECEIVE A 4% INCREASE
IN THEIR PAY LEVEL AND POST
DOCTORAL SCHOLARS WILL INITIALLY
RECEIVE INCREASE OF 8% WITH PAY
LEVELS ADJUSTED UPWARDLY BASED
ON YEARS OF EXPERIENCE AND NIH
AIMS TO INCREASE PAY LEVELS OVER
NEXT FIVE YEARS AND ELIGIBLE
RECIPIENTS WILL RECEIVE A 5
HUNDRED DOLLAR INCREASE IN
SUBSIDIES FOR CHILD CARE AND
ADDITIONAL 2 HUNDRED DOLLARS
RELATED FOR TRAINING AND
EXPENSES AND LOTS OF DISCUSSION
ABOUT HOW TO IMPLEMENT
RECOMMENDATIONS IN CONTEXT OF A
TIGHT BUDGET RELATIVELY MODEST
INCREASES WITH PLAN TO CONTINUE
INCREASES OVER TIME AND
RECOGNIZING IMPORTANCE OF THIS
TOPIC.
HERE AT NIAMS AS PART OF EFFORT
TO MAXIMIZE SCIENTIFIC IMPACT OF
INVESTMENTS AND SUPPORT A BROAD
PORTFOLIO OF MERITORIOUS
RESEARCH GRANTS IN NIAMS MISSION
INSTITUTE ANNOUNCED A NEW
FUNDING POLICY AND NEW POLICY
IMPORTANTLY IS STRONGLY ALIGNED
WITH BROADER NIH GOALS AND
EFFORTS TO BALANCE, STRENGTHEN,
BROADEN AND STABILIZE THE
BIOMEDICAL RESEARCH WORKFORCE
AND EFFECTIVE WITH FISCAL YEAR
2025 APPLICATIONS, ALL NEW AND
RENEWED R01 APPLICATIONS WILL BE
CONSIDERED LOW PROGRAM PRIORITY
IF PI HAS 3 OR MORE ACTIVE R01
EQUIVALENT GRANTS EITHER FUNDED
BY NIAMS OR ANOTHER INSTITUTE
CENTER OR OFFICE AND POLICY WILL
APPLY TO R01 APPLICATIONS FROM
MULTIPLE PIS IF ONE HAS THREE OR
MORE ACTIVE R01 EQUIVALENT
GRANTS AND EXEMPTIONS INCLUDE --
NIAMS FUNDED R01 OR IF ONE OR
MORE EXISTING ACTIVE GRANTS WILL
EXPIRE IN LESS THAN A YEAR
RESULTING IN 2 OR FEWER ACTIVE
R01 EQUIVALENT GRANTS OR
EXISTING R01 ACTIVE EQUIVALENT
GRANTS ARE FROM INSTITUTE
CENTERS AND OFFICES THAT WOULD
BE FIRST NIAMS GRANT.
I ALSO JUST WANT TO MAKE THE
GENERAL COMMENT THAT THIS IS OR
HAS BEEN DEEPLY DISCUSSED ACROSS
NIH WITH A CONSENSUS THAT THIS
IS AN IMPORTANT -- THESE SORTS
OF EFFORTS ARE GOING TO BE
IMPORTANT DURING THESE TIGHT
BUDGETARY TIMES TO STAGIZE
RESEARCH WORKFORCE.
WE WERE, YOU KNOW, PLEASED TO BE
ABLE TO ADOPT THIS POLICY
STARTING IN THE COMING YEAR.
LAST BUT NOT LEAST NIAM PLAN
FISCAL YEARS 20205 TO 2020 KNIFE
AND WILL HEAR LATER TODAY FROM
STEPHANIE BUREAUS FROM A POLICY
AND PLANNING BRANCH THIS
AFTERNOON AND WILL KEEP MY
REMARKS ABOUT THIS BRIEF AND
REMIND YOU THAT NIAMS STRATEGIC
PLANS ARE IMPORTANT BECAUSE THEY
REPRESENT A PUBLIC STATEMENT OF
THE INSTITUTE'S INTEREST, THEY
SEFB AS A GUIDE POST FOR THE
SCIENTIFIC COMMUNITY AND AS AN
INTERNAL REFERENCE DOCUMENT AND
WE HAVE THEREFORE MADE A
CONCERTED EFFORT TO CANVAS THOSE
INTERESTED PROVIDING THEM
OPPORTUNITIES TO PROVIDE INPUT
AND INCLUDING IN SEPTEMBER OF
LAST YEAR OVER 160 RESEARCHERS
A
AS WELL AS STAFF FROM INSTITUTE
AS CROSS NIH AND PATIENT
REPRESENTATIVES AND OTHERS
JOINED TO SHARE IDEAS ON
CROSS-CUTTING TOPICS THAT
INSTITUTE SHOULD PURSUE
THROUGHOUT THE END OF THE
DECADE.
LAST FALL INSTITUTE ISSUED
ANOTHER REQUEST FOR INFORMATION
TO GET FURTHER INPUT ON SUBSET
OF TOPICS IDENTIFIED IN PROCESS
AND SUGGESTIONS WE HAD RECEIVED
AS PART OF THE STRATEGIC
PLANNING PROCESS.
STAFF NOW REVIEWED INPUT
RECEIVED AND PREPARED A DRAFT
STRATEGIC PLAN FOCUSING ON
THEMATIC RESEARCH OPPORTUNITIES
ALLOWING INSTITUTE TO MAKE A
DIFFERENCE IN THE LIVES OF
IDEALLY ALL PEOPLE LIVING IN
AMERICA AND SHARED DRAFT PLAN
WITH MEMBERS OF ADVISORY COUNCIL
FOR THEIR REVIEW PRIOR TO THIS
MEETING.
OF COURSE, AS I MENTIONED
STEPHANIE BUREAUS WILL PRESENT
THE DRAFT PLAN AND WELCOME INPUT
AND COMMENTS ABOUT THAT AT THAT
TIME AND THAT CONCLUDES MY
COMMENTS.
I WOULD BE HAPPY TO TAKE ANY
QUESTIONS YOU MAY HAVE. I WILL
GO DOWN AND SIT AROUND THE
TABLE.
EVERYTHING IS OPEN. QUESTIONS
ABOUT MY COMMENTS AND ABOUT NEW
TOPICS BUT WOULD LIKE TO DEFER
SUBSEQUENT DISCUSSION ABOUT
STRATEGIC PLAN UNTIL AFTER
STEPHANIE'S PRESENTATION.
>> LINDSEY COMING BACK TO THE
TABLE NEWEST COUNSELOR KARL
JEPSON JOINED US AND WOULD LIKE
TO GIVE HIM A CHANCE TO
INTRODUCE HIMSELF.
>> GOOD MORNING. THANK YOU FOR
THE INTRODUCTION AND ALL OF THE
KIND WORDS I APPRECIATE T I'M A
NEW MEMBER OF THE COUNCIL TEAM
AND HAPPY TO BE HERE AND I'M A
PROFESSOR OF ORTHOPEDIC SURGERY
AND BIOLOGIC [INDISCERNIBLE] AND
RESEARCH FOR MEDICAL SCHOOL.
>> WELCOME AND THANKS SO MUCH
FOR JOINING US AND SORRY IF I
MISSED YOU EARLIER ON I WAS
SCANNING AND LOOKING AROUND THE
SQUARES HERE.
SO, WE HAVE SET ASIDE ANOTHER 20
MINUTES TO TAKE QUESTIONS AND
ANSWERS AND ANY DISCUSSION
TOPICS.
DARREN TURNED OFF MY CAMERA.
LET ME TURN IT BACK ON.
HELLO, EVERYBODY.
OKAY. HAPPY TO TAKE YOUR
QUESTIONS OR COMMENTS OR NEW
TOPICS FOR DISCUSSION AT THIS
TI
TIME.
WE WILL KEEP EYE ON BOARD IN
FRONT OF US AND THOSE
PARTICIPATING ONLINE, PLEASE
RAISE YOUR ZOOM HAND AND PICTURE
WILL MOVE TO UPPER LEFT CORNER
HERE.
WE WILL BE ABLE TO SEE THAT.
>> I LIKED HEARING ABOUT THE
COMPLEMENTARY PROGRAM AND THINK
IT RESONATES WELL WITH SOME
THINGS IN THE FDA MODERNIZATION
ACT THAT WERE SIGNED IN 2022
THAT IS TO SAY THAT YOU CAN
ADVANCE A PROGRAM OF DRUG
DEVELOPMENT AND DON'T NEED TO
USE ANIMAL MODELS.
AS SOMEBODY THAT SPENT A LOT OF
TIME REVIEWING AND ON PANELS AND
THINGS LIKE THAT, I'M STRUCK
THAT WE ARE -- WE HAVE LOTS OF
EDUCATION TO DO WITH REVIEWING
PANELS AND WILL SAY I'M EDITOR
OF A JOURNAL AND SEE AMONG
REVIEWERS THERE WE HAVE TO DO
EDUCATION PROGRAM AND GET PEOPLE
TO REALLY UNDERSTAND WHAT ARE
DIFFERENT OPPORTUNITIES THAT ARE
ALTERNATIVES THAT ARE
COMPLEMENTARY TO ANIMAL
RESEARCH? I FEEL WE ARE IN
DEFICIENCY AND LOTS OF REVIEWERS
NOW OF NOT REALLY UNDERSTANDING
WHAT ARE PITFALLS OF USING SOME
APPROACHES AND WHAT IS THE POWER
OF USING THOSE AND DON'T KNOW IF
THERE HAS BEEN DISCUSSION OR
THOUGHT ABOUT THAT PROCESS HOW
WE CAN DO BETTER AND KNOW FROM
MY END AS A JOURNAL EDITOR I'M
THINKING ABOUT HOW TO GET PEOPLE
TO WRITE POSITION PIECES AND
IMPROVE EDUCATION ON THIS.
RIGHT NOW THERE IS A REAL
DISCONNECT IN TERMS WHAT IS
GETTING REVIEWED AND WHAT IS
POSSIBLE.
>> THANKS FOR THAT COMMENT.
MAKES A LOT OF SENSE AND
APPRECIATE YOU HIGHLIGHTING THAT
AND I'M THINKING THAT I WONDER
WHAT OPPORTUNITIES EXIST THROUGH
CSR IF THAT BECOMES AN APPARENT
NEED, PARTICULARLY WITH THIS NEW
EMPHASIS FOR SOME TRAINING.
YOU KNOW, I MEAN, CSR SECTION
MEMBERS RECEIVE TRAINING IN A
NUMBER OF AREAS AND MIGHT BE AN
IDEA THERE AND DON'T KNOW IF
KATHY IS WITH US BUT, KATHY HAVE
ANY COMMENTS ABOUT THIS IN TERMS
OF A PRECEDENT FOR PROVIDING IN
A SENSE KIND OF EDUCATIONAL OR
OTHER INFORMATION ABOUT NEW
AREAS OF SCIENCE THAT IS
INCREASINGLY RELEVANT TO
APPLICATIONS WE ARE SEEING FOR
WHICH REVIEWERS, YOU KNOW, MAY
NOT BE, YOU KNOW, AS INFORMED
ABOUT?
>> I'M THINKING TISSUE
ENGINEERING AND THINGS ALONG
THOSE LINES.
>> WHAT KATHY MIGHT BE SAYING IS
IT IS A GENERAL PHENOMENON.
>> YEAH.
>> NOTTUS REVIEW YOU ELUDED TO.
>> YEAH.
>> I DO ACTUALLY TAKE YOUR POINT
THAT THESE ARE NEW WAYS OF DOING
THINGS AND HOPEFULLY NOT
SPEAKING FOR MYSELF AND SOME
PEOPLE HAVE A HARD TIME SHIFTING
TO NEW WAYS OF DOING THINGS.
I DO THINK THAT THIS IS A MORE
GENERAL ISSUE.
THERE IS A LOT OF ROOM CERTAINLY
IN -- YOU SAW IDEAS THAT WE
SUPPORT TISSUE ON CHIMP RESEARCH
AND SUPPORTED STUDIES OF OA ON A
CHIP AND OSTEOARTHRITIS AND A
CHIP AND I WILL SAY SPEAKING
FROM MYSELF TO SOMETIMES IT IS
HARD TO REALLY KNOW WHAT
STRENGTHS AND WEAKNESSES AND
APPROACHES ARE.
I DON'T HAVE A GOOD ANSWER.
I COMPLETELY AGREE WITH YOUR
POINT.
>> GREAT. TED HAS A COMMENT
HERE.
>> YEAH. RECENTLY WE PUBLISHED
NO SEE WITH NATIONAL INSTITUTES
OF AGING AND PURPOSE OF NO SEE
IS ENCOURAGE COMMUNITY TO USE
AND UTILIZE MODEL TO STUDY
OSTEOARTHRITIS AND EMPHASIZE
TISSUE AND CHIMP CELL MODEL
OTHER THAN TRADITIONAL ANIMAL
MODEL.
>> THANK YOU. THIS IS GREAT TO
HAVE MANY NIAMS REPRESENTATIVES
AND NO SEE REFERS TO NOTICE OF
SPECIAL INTEREST AND IMAGINE
THAT THERE WILL BE GREAT
PUBLICATIONS THAT COME OUT AS A
RESULT OF WORK OF
COMPLEMENTARYIAN EFFORTS AND
SEEING EARLY ADOPTERS PUTTING IN
APPLICATIONS AND NOT FAREWELL
BECAUSE OF TRADITIONAL THINKING.
ANYWAY, YEAH. IT WOULD BE
INTERESTING TO FIND OUT WHAT
PLANS THERE ARE FOR BROADER
COMMUNICATION AND EDUCATION OF
THE COMMUNITY AND THANK YOU FOR
HIGHLIGHTING THAT.
>> DO YOU HAVE SUGGESTIONS?
>> YEAH.
>> WELL, I MEAN, WE HAVE
MUSCULAR DISTROPHY ASSOCIATION
GRANT REVIEWS NEXT WEEK AND ASK
TO PUT ASIDE TIME TO TALK ABOUT
THIS AT GRANT REVIEW PANEL TO
EDUCATE PEOPLE AND WEARING
JOURNAL EDITOR HAT THAT IS WHERE
WE CAN PROBABLY MAKE BIGGEST
DIFFERENCE THAT IS MAKING IT
CLEAR THAT WE DO -- WE DON'T
NEED TO DO EVERYTHING IN MOUSE.
HUMANS ARE LARGELY TRYING TO
MODEL AND HUMAN CELL MODELS,
THERE IS VALUE TO THAT AND ALONG
WITH THAT COMES RIGOR AND
REPRODUCIBILITY AROUND HUMAN
VARIABILITY THAT BECOMES HARDEST
PART ABOUT DOG HUMAN CELL MODEL
AND WE HAVE LOTS TO DO AND
TEACH.
>> GREAT.
>> DR. [INDISCERNIBLE].
>> KEY EXAMPLE OF THAT WOULD BE
HOW NIH IS NOW TRAINING OR
ADVISE REVIEWERS AND WE HAVE PUT
OUT NEW PROGRAMS TO INFORM
REVIEWERS ON CHANGING HOW WE
REVIEW IT.
WE HAVE DONE EFFECTIVE PROGRAMS
WITH THAT AND IT WOULD BE A
GREAT IDEA TO MAKE MODIFICATIONS
TO CSR DUE TO A SIMILAR
TRAINING.
>> YEAH. THANK YOU. WE WILL
BRING A CHAIR OVER SO WE DON'T
WANT ANY INJURED KNEES AT END OF
THE COUNCIL MEETING.
SO YEAH. MARIA?
>> YEAH. THANK YOU, NANCY FOR A
WONDERFUL PRESENTATION AND
DELIGHTED TO SEE NEW INITIATIVES
MOVING FORWARD AND WONDERING IF
YOU COULD ELABORATE A LITTLE BIT
ON PROCESS TO RECOMMEND FUNDING
FOR THOSE APPLICATIONS THAT WILL
BE ABOVE THE PAY LINE.
>> YEAH. SO I GUESS ONE THING I
WILL CLARIFY IS WE ARE CALLING
THAT PROCESS SELECT PAY
COMPLEMENT TO IT IS
COMPLEMENTARY TO THE PAYLINE
DRIVEN APPROACH AND NIAMS HAD A
SELECT PAY IN SENSE THAT AT END
OF EVERY FISCAL YEAR FUNDS FLOW
BACK FROM INSTITUTE AND
INVESTIGATOR LEAVES ACADEMIC
INSTITUTE OR WHATEVER AND CAN'T
CARRY FORWARD FUNDS AND HAVE TO
SPEND EVERY PENNY WE HAVE
OTHERWISE WE HAVE A PROBLEM AND
ALWAYS AT END OF THE FISCAL
YEAR, APPLICATIONS WERE
IDENTIFIED FOR SELECT PAY
TYPICALLY ON BASIS OF AN EARLY
CAREER INVESTIGATOR AND AT-RISK
INVESTIGATOR AND HIGH PRIORITY
AREA BIG GAP IN PORTFOLIO AND
THAT SORT OF THING AND
UNPREDICTABLE AND SMALL AMOUNT
OF FUNDS ONLY AT END OF THE
FISCAL YEAR AND WAS A SCRAMBLE
BECAUSE OF THE LAST-MINUTE
NATURE OF IT AND NOW WE ARE
SPREADING OUT SELECT PAY TO 3
TIMES A YEAR FOLLOWING EACH
COUNCIL MEETING.
YOU ALL SIGN OFF ON THE SLATE OF
APPLICATIONS AND THE DECISIONS
ARE MADE IN THE CONTEXT OF TEAMS
OF RELATED PROGRAMS, SKIN OR
MUSCLE OR ARTHRITIS ON BASIS OF
INVESTIGATOR AND IS IT EARLY
CAREER INVESTIGATAR OR AT-RISK
INVESTIGATOR WHOSE LAB MAY CLOSE
DOWN WITHOUT A FUNDED
APPLICATION AND DOES IT ADDRESS
A PRIORITY AREA WE HAVE
HIGHLIGHTED IN STRATEGIC PLAN OR
NOTICE OF SPECIAL INTEREST AND
GAP AREA WE IDENTIFIED.
THOSE ARE THE MAIN CATEGORIES
AND AM I MISSING A MAJOR
CATEGORY?
THOSE ARE MAIN THINGS.
YEAH. WE WANT TO EMPHASIZE THE
FACT THAT, YOU KNOW, KIND OF THE
PORTFOLIOS ACROSS INSTITUTE ARE
SOMEWHAT DIFFERENT AND SOME
PORTFOLIOS HAVE A POSITY OF
EARLY CAREER INVESTIGATORS AND
PROGRAM MANAGERS ARE THINKING
ABOUT MAKING SURE WE CONTINUE TO
HAVE INVESTIGATORS MOVING INTO
THE FIELD AND OTHER AREAS HAVE
DIFFERENT ISSUES AND MAY BE BIG
GAPS IN PORTFOLIO AND POSITY OF
FUNDING FOR VARIOUS REASONS AND
HAVEN'T BROUGHT IN RIGHT
INVESTIGATORS AND MIGHT BE
MISSING RIGHT TOOLS WE NEED AND
MIGHT BE A CONCERN ABOUT THE
REVIEWED PROCESS AND THOSE
APPLICATIONS NOT BEING REVIEWED
CAREFULLY.
SO THOSE ARE THINGS AND OF
COURSE SCORE IS CONSIDERED AS
PART OF SELECT PAY AS WELL.
WE HAVE QUESTIONS ABOUT HOW YOU
WILL MONITOR THIS AND ASSESS IT
AND DEVELOP METRICS GROUP
INCLUDING FOLKS FROM BUDGET AND
PROGRAM LEADERSHIP TO TRY TO
IDENTIFY METRICS ALLOWING US TO
FOLLOW OUR BROADLY OUR FUNDING
APPROACH AND WHAT IS HAPPENING
WITH SELECT PAY AND WHAT ARE
IMPACTS OF THE NEW POLICY
RELATED TO INVESTIGATORS WITH
MORE THAN THREE REWARDS AND ABLE
TO MAINTAIN SAME NUMBER OF
AWARDS FROM YEAR TO YEAR AS
BUDGETS EVOLVE.
>> METRICS AFTER AWARDS HAVE
BEEN GIVEN, I GUESS.
I WAS WONDERING AS FAR AS THE
SELECTION OF THOSE GRANTS, IT
HAS BEEN -- I MEAN, IN THE PAST
IT HAS BEEN QUALITATIVE WITH
REMAINING FUNDS AS YOU MENTIONED
AND THAT PROCESS IS NOT
CHANGING.
IT WILL BE QUALITATIVE
ASSESSMENT OF WHAT THE NEEDS.
>> WELL, THIS -- THIS -- THE
IDENTIFIED -- SO, THE GRANTS GET
REVIEWED AND SCORES COME TO US
AND THEN WE ARE EVALUATING
BUDGET AND WHAT PAY LINE CAN BE
AND WE ARE SETTING ASIDE FUNDS
FOR EACH ROUND FOR SELECT PAY
AND INDIVIDUAL PROGRAM OFFICERS
ARE WORKING IN TEAMS NOMINATE
APPLICATIONS FOR SELECT PAY ON
BASIS OF THOSE CRITERIA AND WE
DON'T HAVE A STRICT FORMULA.
WE THINK WE NEED TO HAVE FLEX
INLT ACROSS PORTFOLIOS AND IS
HARD TO -- WHAT IS IMPORTANT?
THERE IS NOT A STRICT FORMULA
AND THERE IS CLEAR
CONSIDERATIONS THAT ARE TEAMS
THAT CONSIDER IN NOMINATING
APPLICATIONS FOR SELECT PAY.
>> MY ONE CONCERN IS ON PART OF
INVESTIGATORS IF MORE BUDGET
WILL GO TO SELECT PAY THAN IT
WAS BEFORE AS PERCENT OF ALL OF
THE FUNDS MIGHT BE INTEREST IN
MORE TRANSPARENCY AS TO HOW
THOSE DECISIONS ARE MADE.
I WANTED TO COMMENT, YEAH.
>> YEAH. I GUESS, YOU KNOW, THE
-- I -- LET ME KNOW. I'M NOT
SURE WHAT YOU MEAN IN TERMS OF
TRANSPARENCY.
PROCESS IS APPLICATIONS ARE
REVIEWED AT CSR.
AND THEN SCORES COME TO US AND
THEY GET DIVIDED INTO TEAMS
BASED ON TOPIC AREA.
WE ARE INTERACING WITH BUDGET TO
FIND OUT HOW MUCH MONEY WE HAVE
AND TEAMS SIT DOWN AND GO
THROUGH APPLICATIONS THAT DON'T
NEED PAY LINE AND CONSIDER ARE
ANY OF THESE INVESTIGATORS AT
RISK?
ARE ANY EARLY CAREER
INVESTIGATORS OR AT-RISK
INVESTIGATORS OR ANY
APPLICATIONS IN RESPONSE TO NO
SEES WE PUBLISH OR ARE
ADVERTISING THAT ARE IN INTEREST
OF THIS AREA?
I WILL BASE IT ON THAT AND THEY
ARE CHALLENGING DECISIONS TO
MAKE.
MAIN PROBLEM WE HAVE IS WE HAVE
RELATIVELY MODEST FUNDS THAT ARE
COMPARED TO OUR MISSIONARIES AND
I VIEW -- I QUOTED THIS FIGURE
MANY TIMES.
ROUGHLY 3% OF R01 APPLICATIONS
SENT TO THE NIH COME TO NIAMS
AND BUDGET ALLOCATIONS ARE MORE
THAN 1.5%.
WE ARE CHALLENGED RIGHT OFF THE
BAT WITH HAVING TOO MANY STRONG
APPLICATIONS THAT WE WOULD LIKE
TO FUND THAT WE -- WE CAN'T.
AND WE THINK THAT BECAUSE OF
THOSE VERY SIGNIFICANT
STRENGTHS, IT SPEAKS MORE TO THE
NEED FOR SELECT PAY TO HAVE SOME
FLEX INLT AND OTHERWISE CSR IS
BASICALLY MAKING ALL DECISIONS
FOR US IF WE JUST USE THE SCORE.
>> I MEAN, I THINK IT WOULD BE
USEFUL TO HAVE AND YOU WOULD
HAVE IT ALREADY PUBLISHED AND
HAVE A LIST OF CRITERIA THAT ARE
USED FOR THIS AND THEY ARE
PUBLISHED. YOU KNOW, WHEN THE
AWARD IS FUNDED, EVEN THE
INVESTIGATOR KNOWS THR01 WAS
FUNDED BECAUSE OF THIS, THIS,
AND THAT AND THAT DECISION.
>> I SECOND THAT AND WHAT MARIA
WAS SAYING AND SAY THAT SINGLE
BIGGEST THING I HAVE HAD PEOPLE
ASK ME ABOUT IN LAST YEAR
KNOWING I'M ON COUNCIL IS
EXACTLY THIS TOPIC AND I'M SURE
EVERYONE IN THE ROOM WILL SAY
SAME THING.
MY GOODNESS.
THEY ARE DOING SELECT PAY.
I SAY IT IS A VERY SMALL AMOUNT
OF BUDGET THAT IS MEANT FOR
THESE THINGS AND IF YOU
REITERATE THAT MESSAGE AND MAKE
IT CLEAR TO PEOPLE IT WILL DO A
LOT TO CALM THE COMMUNITY.
>> LET ME ADD IN THAT WE
CERTAINLY, YOU KNOW, PEOPLE GET
A GRANT AWARD AND ARE ENCOURAGED
TO TALK TO THEIR PROGRAM OFFICER
AND ASK THEM ABOUT THAT.
WE ARE LIMITED INTO DATA WE CAN
PUBLISH. WE ARE HAPPY TO
PUBLISH AGGREGATE DATA AND CAN'T
PUBLISH DATA SAYING WE FUNDED
THIS GRANT FOR THESE SELECT PAY
REASONS, FOR INSTANCE.
THAT CAN BE -- THAT IS
CONFIDENTIAL.
WE WILL -- WE DO HAVE PLANS TO
PUBLISH A LOT OF DATA GOING
FORWARD AND WOULD LIKE TO HEAR
YOUR FEEDBACK ABOUT THAT.
>> YOU KNOW, CORRECT ME IF I'M
WRONG.
I BELIEVE WE HAVE ALREADY
CLARIFIED ON OUR WEBSITE THE
CONSIDERATIONS THAT APPLY FOR
SELECT PAY.
YOU KNOW, WE DON'T SAY YOU HAVE
TO BE AN EARLY CAREER
INVESTIGATOR OR ONLY THIS MANY.
WE -- YOU KNOW, IT IS -- IT IS
THESE RELATIVELY BROAD
CATEGORIES.
YOU KNOW, WE DON'T THINK A
HIGHLY PRESCRIPTIVE FORMULAIC
APPROACH WILL WORK. YOU KNOW?
JEN EES HAS HER HAND UP AND HAS
A COMMENT ON THIS TOPIC AS WELL.
>> THANK SO YOU MUCH, LINDSEY,
FOR YOUR PRESENTATION.
I APPRECIATE THIS DISCUSSION.
JUST TWO QUICK QUESTIONS.
THE FIRST ONE IS WHAT PERCENT OF
THE BUDGET ARE YOU ANTICIPATING
WILL BE PART OF SELECT PAY?
I KNOW YOU MENTIONED
FLEXIBILITY.
IS THERE A PERCENTAGE THAT HAS
BEEN THROWN AROUND.
>> YEAH.
>> SECOND QUESTION, WHAT
INTEGRAL WILL YOU LOOK AT
METRICS AND SORT OF DECIDE WHAT
WILL HAPPEN IN THE NEXT YEAR?
>> SO PERCENT WE SET ASIDE IS
20%.
80% GOES TO SUPPORT PAY LINE.
20% FOR SELECT PAY AND, OF
COURSE, YOU KNOW, IF -- YOU
KNOW, OF COURSE THE 8% RELATES
WHOLLY TO THE AMOUNT OF MONEY
THAT FLOWS TO NIAMS RELATIVE TO
APPLICATION NUMBERS ARE YOU
SEEING AND ALSO PROGRAM OFFICERS
ARE YOUR PALS FOR INVESTIGATORS
AND ARE HERE TO SERVE THEM.
WHEN I TALK TO THEM, THEY FIND
THAT MOST GRATIFYING AND THEY
LOVE SCIENCE AND WORKING AT NIH
AND WANT TO HELP INVESTIGATORS
AND REASON TO ENCOURAGE
INVESTIGATORS TO REACH OUT TO
PROGRAM OFFICERS IS THEY ARE
WELL AWARE OF SCIENTIFIC OVERLAP
AND AVAILABILITY AT INSTITUTES
AND CENTERS AND APPLICATION THAT
SURE CAN GO TO NIAMS AND ALSO
COULD GO TO NIA AND IF THEY
HAPPEN TO HAVE A HIGHER PAY LINE
OR SPECIAL INTEREST THEY WILL
ENCOURAGE THEM.
WE WANT TO ENCOURAGE
INVESTIGATORS TO DIVERSIFY
FUNDING. YOU KNOW, CERTAINLY
OUTSIDE OF THE NIH TO MAKE SURE
THEY ARE FULLY LEVERAGING OTHER
FUNDING SOURCES AND ALSO TO
FULLY LEVERAGE OPPORTUNITIES
ACROSS THE NIH AND LUPUS FOR
EXAMPLE IS RELEVANT TO 6 OR 8.
ICS.
ALSO, OF COURSE, FUNDING
OPPORTUNITIES COME UP THROUGH
HEEL AND HELPING TO END
[INDISCERNIBLE] LONG TERM AND
OTHERS AND PROGRAM OFFICERS,
THIS COMES UP ALL THE TIME WE
GET AN INQUIRY AND APPLICATION
ABOUT PAYING AND ARE YOU AWARE
OF HEEL FUNDING OPPORTUNITIES
AND WE ARE LOOKING FOR
OPPORTUNITIES TO PRESERVE OUR
BUDGET AS WELL.
SO, THE 20% IS WHAT WE HAVE SET
ASIDE FOR SELECT PAY AND WE WILL
CONTINUE TO MONITOR THAT.
NOW, IN TERMS OF METRICS, I
SUSPECT WE WILL LOOK AT THEM
INTERNALLY THREE TIMES A YEAR
AFTER EACH COUNCIL ROUND AND
REPORTING THEM OUT ANNUALLY AND
WORK, THIS IS A CHANGE.
AND THE ENVIRONMENT IS CHANGING.
YOU KNOW, EVEN IF WE HAVE THIS
PERFECT NOW, IT WON'T BE PERFECT
FOR TOMORROW.
WE WILL CONTINUE TO LOOK AT
METRICS.
WE HAVE LOTS OF INTEREST IN
UTILIZING THE SYSTEMS AND
INFORMATION WE HAVE AVAILABLE TO
LOOK AT IN INDIVIDUAL INVEST --
UNIQUE INVESTIGATORS AND NUMBER
OF GRANTS AND DISTRIBUTION OF
SCORERS.
I WAS ACTUALLY SURPRISED YOU MAY
ALL WANT TO TAKE A LOOK.
ON THIS NOTE, MAYBE IF SOMEONE
CAN PUT IN THE CHAT, I DIDN'T
REALIZE THAT IT WAS PUBLICLY
AVAILABLE INFORMATION THAT YOU
CAN LOOK FOR EACH INSTITUTE AND
CENTER AT THE OVERLAPPING
DISTRIBUTION BETWEEN FUNDED AND
NOT FUNDED AND AWARDED
APPLICATIONS.
THERE IS A BIG OVERLAP.
IN OTHER WORDS, FOR MOST
INSTITUTES.
IN OTHER WORDS, THERE IS A LOT
OF APPLICATIONS THAT -- HOW CAN
I SAY THIS? YOU KNOW, YOU HAVE
FUNDED AND NOT FUNDED AND THERE
IS A BIG OVERLAP THERE.
IT IS NOT JUST A SCORE.
OTHER INSTITUTES AND CENTERS ARE
NOT RELYING EXCLUSIVELY ON THE
SCORE TO A MAYBE SURPRISING TO
YOU EXTENT.
THIS IS NOT.
>> LET ME SAY FOR THOSE THAT
CAN'T SEE THE CHAT.
>> OKAY.
>> DO A SEARCH FOR NIH DATA BOOK
THAT WILL TAKE YOU TO ALL OF
THIS INFORMATION THAT LINDSEY
WAS JUST MENTIONING.
>> WE SPENT A LOT OF TIME AND
EFFORT REACHING OUT TO OTHER
INSTITUTES AND CENTERS TO
UNDERSTAND HOW THEY ARE
APPROACHING THESE ISSUES AND,
YOU KNOW, LESSONS LEARNED.
WHAT ARE THE CHALLENGES
ASSOCIATED WITH, YOU KNOW,
DIFFERENT APPROACHES AND WE
INVESTED TIME AND EFFORT AND WE
UNDERSTAND THIS IS A BIG DEAL
FOR THE COMMUNITY AND WANTED TO
TAKE A THOUGHTFUL APPROACH AND
TRY TO GET IT AS RIGHT AS
POSSIBLE FROM THE OUTSET.
VALENTINA?
>> I WANTED TO CONNECT
CONVERSATION AND ONE ABOUT BEING
A CONVENTION THAT WE HEARD ABOUT
BEFORE AND BASIC BIAS TRAINING
AND SELECT PAY.
I THINK THAT IT -- THESE POCKETS
CAN CREATE AN OPPORTUNITY IF WE
GO AS A SLATE IN PUTTING ON
PRESSURE VALUES THAT WE WANT TO
LEAD THAT WILL BE MUCH BY THE
OUTCOME AND MEAN BY THAT IF WE
WANT TO [INDISCERNIBLE] AND
DISTRIBUTING FUNDING IN A WAY
THAT IS EQUITABLE ACROSS
INSTITUTIONS IN THE UNITED
STATES AND ACROSS IDENTITIES AND
LENS, THERE IS A POSSIBILITY TO
USE SELECT PAY TO COMPLEMENT AND
INTEGRATE IN WAY THAT NUMBERS OF
GRANTS THAT HAVE FUNDING IN THAT
PERIOD IS DISTRIBUTED EQUITABLY
AND LEAVING TO OWN DEVICES WE
ENFORCE FUNDING OF PEOPLE THAT
SOUND LIKE US AND ARE ALREADY
DOMINATING AND WE ARE NOT
CHANGING LANDSCAPE AND SELECT
PAY CAN HELP US JUMP THAT HURDLE
ESPECIALLY COMBINING WITH
IMPLICIT BIAS TRAINING AND PUT
US TO HAVE AN OUTCOME AS MUCH AS
VALUES THAT HAS TO BE DONE
BEFORE IT IS FINALIZED TO
CORRECT AND PERFECTION THAT WE
WILL IMPOSE.
>> GREAT SUGGESTION THAT YOU
REMINDED ME AND ANOTHER CORRECT
ME IF I'M WRONG, FOLKS.
WE DISCUSSED IT CONSIDERING ALSO
THE STATUS OF THE INSTITUTION
AND INSTITUTION WITH FEWER
RESOURCES AND OPPORTUNITIES TO
SERVE NIH FUNDING SERVING FROM
STANDPOINT OF NIH LEADERSHIP IS
A REASON TO GIVE AWARD SELECT
PAY AND EVERYTHING ELSE BEING
EQUAL AND SPREADING THE
OPPORTUNITIES.
>> AND THE KEY IS WHAT YOU SAID
EVERYTHING BEING EQUAL THAT
CANNOT BE EQUAL AND WE ARE NOT
ACCESSING RESOURCES MEMBERSHIP
TO BE ON TO AND INSTITUTION WE
ARE AT CAN'T USE SAME METRICS
THAT ARE MADE WITH PEOPLE THAT
HAD APPROXIMATELY POWER TO
RESOURCES AND THERE IS ATTENTION
I FEEL WHEN WE DEFINE METRICS
AND NIH HAS INCREDIBLE OFFICE
THAT CAN BE TAPPED ON AND
METRICS PUT IN PLACE SO FAR AND
EVOLVED TO BE TRUTHFUL TO WHERE
WE WANT TO GO.
>> YOU ARE SUGGESTING DR. ROBERT
WALKER AND OTHERS PERHAPS
SPEAKING OF UPCOMING COUNCIL
MEMBER AND ARE THINKING DEEPLY
ABOUT ISSUES AND DARREN REMINDED
ME WE ARE A LITTLE OVER INTO THE
NEXT SESSION AND MOVING TO
PATIENT ENGAGEMENT AND WE HAVE
OPPORTUNITIES FOR Q & A IN OPEN
AND CLOSED SESSION AND KEEP
THOUGHTS IN MIND.
NOW, I THINK THAT I'M TURNING TO
BOB.
>> DR. CARTER.
>> THANK YOU. AND REALLY
LOOKING FORWARD TO THIS SESSION
ON THE PATIENT ENGAGEMENT AND I
WILL INTRODUCE THREE SPEAKERS.
ONE OF WHOM I DON'T SEE YET.
SO, PETER, I HOPE HE WILL JOIN
US.
BUT THIS SESSION IS GOING TO BE
KICKED OFF BY SUE TRANWHO IS A
PATIENT AND PATIENT ENGAGEMENT
ACTIVIST WITH SDANLT LAW AND
POLICY BACKGROUND WHO IS
RECENTLY A MEMBER OF COUNCIL AND
RECEIVED HER LAW DEGREE FROM
UNIVERSITY OF KANSAS AND CAREER
SPANS WORK IN BIOETHICS
DISABILITY RIGHTS GENETIC
DISCRIMINATION AND HISTORY OF
PATIENT LED HEALTH EDUCATION AND
FOUNDER AND CEO AND EXPECT
PATIENT ENGAGEMENT AND PUT IN
DUE TANK AND SERVED IN
ENGAGEMENT LEADERSHIP ROLES
PATIENT CENTERED OUTCOME
RESEARCH INSTITUTE AND
AUTHORIZED FOUNDATION AND SERVES
ON FDA'S PATIENT ADVISORY
COMMITTEE AND IMMEDIATE PAST
CHAIR OF INTERNATIONAL SOCIETY
OF ECONOMICS AND OUTCOMES
RESEARCH AND GLOBAL PATIENTS
COUNCIL AND CO-AUTHORED MULTIPLE
PEER REVIEWS AND VALUE OF
PATIENT ENGAGEMENT AND SESSION
IS HONOR AND ROLE AND ADVOCATE
DURING TENURE AND ROLE ON
COUNCIL AND PROMISED THIS
SESSION THAT ARE FITTING WITH
TALKS SHE WILL HEAR LATER TODAY
ON CLINICAL STUDIES AND WE ARE
DELIGHTED TO COMPLETE THE
PROMISSORY NOTE WE GAVE YOU TO
MONTHS AGO.
THANK YOU FOR JOINING AND
LEADING OFF.
>> THANK YOU SO MUCH AND FOR THE
OPPORTUNITY TO BE HERE AND
PRESENT AND SPECIAL THANKS TO
YOU DR. CARTER AND SO SAD TO SEE
YOU GO. PRIVILEGE AND HONOR TO
WORK WITH YOU IN THIS CAPACITY.
I WILL MOVE REALLY QUICKLY
THROUGH MY SLIDES.
I KNOW WE HAVE LIMITED TIME AND
I WILL BREEZE OVER A COUPLE.
YOU HAVE THEM IN YOUR COUNCIL
BOO
BOOKS.
I THINK SOME -- YEAH. THERE WE
GO.
I WILL FOCUS ON VERY HIGH-LEVEL
KEY POINTS AND WILL START WITH
THE WHAT OF THE -- YOU KNOW,
WHAT IS ENGAGEMENT AND WHY DO WE
DO IT AND SPEND REMAINDER OF
TIME FOCUSED ON HOW AND REALLY
TRYING TO GET TANGIBLE AND
TECHNICAL.
LET'S GO TO THE NEXT SLIDE AND
NEXT SLIDE AND PAUSE.
GREAT. THANK YOU.
BIGGEST CHALLENGE IN PATIENT
ENGAGEMENT IS TERM PATIENT
ENGAGEMENT THAT MEANS SO MANY
THINGS TO SO MANY DIFFERENT
PEOPLE AND SMALL GROUP OF US
THAT STARTED TO ELEVATE
BIFURCATED LANGUAGE AROUND TYPE
A AND TYPE B ENGAGEMENT AND TYPE
A ENGAGEMENT IS WHAT REALLY MOST
PEOPLE MEAN AND MORE TRADITIONAL
NOTION OF PATIENTS BEING ENGAGED
IN OWN CARE AND OWN DISEASE
MANAGEMENT AND ENGAGED IN THEIR
OWN INVOLVEMENT IN CLINICAL
TRIALS AS STUDY SUBJECTS AND
PARTICIPANTS AND TYPE B PATIENT
ENGAGEMENT THEN IS A NEWER PECK
ORRI MODEL OF ENGAGEMENT NOT
ABOUT PATIENTS BEING INVOLVED IN
THEIR OWN CARE OR OWN
INVOLVEMENT AND RESEARCH AND
ABOUT PATIENTS SERVING AS
PARTNERS ADVISORS LEADERS AND
SITTING SIDE BY SIDE WITH
RESEARCHERS AND CLINICIANS IN
CODESIGN AND CO-CONDUCT AND
CO-DISSEMINATION OF RESEARCH AND
HEALTH CARE INNOVATION.
TWO TYPES ARE BOTH IMPORTANT.
IF WE GO TO THE NEXT SLIDE, WE
CAN REALLY LOOK AT VALUE OF TYPE
A ENGAGEMENT AND THIS IS THINGS
LIKE SHARED DECISION MAKING AND
CO-PRODUCTION OF HEALTH OUTCOMES
PATIENT EDUCATION AND
PEER-TO-PEER SUPPORT AND PATIENT
ENROLLED IN CLINICAL RESEARCH
STUDIES IT IS SUPPORT AND
NURTURING OF PATIENTS TO HAVE
QUESTIONS ANSWERED AND FEEL
COMFORTABLE WITH RESEARCH.
WAY WE GET TO TYPE A ENGAGEMENT
IS THROUGH TYPE B METHODS AND
WAY YOU DESIGN CLINICAL TRIAL TO
BE FEASIBLE AND ACCESSIBLE FOR
STUDY PARTICIPANTS IS PARTNERING
WITH PATIENTS FROM COMMUNITY OF
INTEREST TO MAKE THAT WAY AND
REASON I STARTED WITH THIS IS
YOU WOULD BE AMAZED OR MAYBE YOU
HAVE ALREADY EXPERIENCED THIS,
HOW FAR YOU CAN GET INTO A
CONVERSATION OR INTO AN ENTIRE
WORK STREAM BEFORE YOU REALIZE
THAT HALF OF THE GROUP WAS
TALKING ABOUT TYPE A ENGAGEMENT
AND HALF TALKING ABOUT TYPE B
ENGAGEMENT AND ARE COMPLETELY
DIFFERENT AND REQUIRE DIFFERENT
SKILLS AND TOOLS AND WANT TO LAY
THAT GROUNDWORK FIRST.
LET'S GO TO THE NEXT SLIDE AND
NEXT ONE AND I WILL STOP HERE.
SPENDING A COUPLE MINUTES ON WHY
AND THIS TOUCHES ALSO A LITTLE
ON THE HOW.
THIS IS A STUDY DONE BY BENNETT
LEVITAN AND GET AZ FEW YEARS AGO
AND THEY WANTED TO PUT A DOLLAR
AMOUNT ON VALUE OF PATIENT
ENGAGE PT AND SPECIFIC TO
PATIENT ENGAGE PT AND MEDICAL
PRODUCT R & D AND THEY
CALCULATED FOR EVERY $100,000
INVESTED RETURN ON INVESTMENT
WAS 5 HUNDRED TIMES THAT AMOUNT
AND THIS IS BECAUSE OF REDUCTION
AND PROTOCOL AMENDMENTS AND
REDUCTION OF FAILED TRIALS DUE
TO LACK OF RECRUITMENT AND
RETENTION AND A MYRIAD OF
REASONS.
THIS IS A DOLLARS AND CENTS WAY
TO THINK ABOUT IT THIS IS
LOGICAL FOR ALL KINDS OF
RESEARCH TO HAVE THOSE MEMBERS
OF THE COMMUNITY OF INTEREST AT
THE TANL.
IF WE GO TO THE NEXT SLIDE,
THESE ARE A LITTLE MORE NEAR AND
DEAR TO MY HEART IN RHEUMATOLOGY
SPACE AND EXAMPLE WHAT IT LOOKS
LIKE WITH PATIENTS IN THE ROOM
HELPING PARTNER AND IN
COMPLICATED PARTS OF RESEARCH
AND WAS A STUDY LOOKING AT
TREATMENT TRENDS AND LOOKING AT
CLAIMS DATA AND DATA SCIENTISTS
WERE CONCERNED THAT DATA ITSELF
WAS DEFECTIVE AND THEY SAID WE
ARE SEEING ALL THESE STOPS AND
STARTS OF THERAPY AND PEOPLE
SWITCHING BACK TO A THERAPY THAT
THEY DISCONTINUED AND BLENDING
THERAPIES.
IT LOOKS LIKE A MESS.
WE ARE CONFUSED ABOUT WHY IT
LOOKS THIS WAY.
WHEN BRINGING TO PATIENT
PARTNERS ON STUDY, WE SAID, OH,
YOU KNOW, FIRST OF ALL, CLAIMS
DATA IS NEVER PERFECT.
THIS IS REFLECTIVE OF PATIENT
EXPERIENCE AND WE HAVE TO STOP
FOR SURGERIES, INFECTIONS
INSURANCE REASONS AND SWITCH
BACK TO OLD THERAPY AND THERE
ARE SO MANY REASONS THESE CHANGE
AND WHAT IS CHILLING ABOUT THAT
EXAMPLE IS IT MAKES ME THINK HOW
MANY ROOMS IS THIS DATA ANALYSIS
HAPPENING IN WHERE THERE ARE NOT
PATIENTS OR PRACTICING
CLINICIANS YOU CAN FRAME IT IN
REALITY WHAT IT REALLY MEANS TO
TAKE THERAPEUTICS AND MANAGE
THESE DISEASES AND WE GO TO NEXT
SLIDE.
THIS IS ABOUT LOOKING AT FAR,
FAR END OF THE RESEARCH PARADIGM
AND THINKING ABOUT
IMPLEMENTATION AND UPTAKE OF
FINDINGS AND WAS, AGAIN, FROM A
FEW YEARS AGO WHEN PANEL OF
PATIENTS AND PARENTS
PARTICIPATED IN THE UPDATE OF
JIA CLINICAL PRACTICE GUIDELINES
I WAS AT ARTHRITIS FOUNDATION AT
THAT TIME AND DID ONBOARDING AND
PREP FOR PANEL OF FOLKS AND
BASIC INFORMATION ABOUT HOW
EVIDENCE IS GRADED AND WHAT IT
LOOKS LIKE TO DEVELOP A PICO
STATEMENT AND HOW IT WAS GOING
TO WORK AND ONE OF THE
INTERESTING THING WAS AT THE END
OF ALL THIS EVEN THOUGH ONE OF
THE THERAPIES THAT IS STILL A
GOLD STANDARD THERAPY IS
RELATIVELY SAFE AND RELATIVELY
EFFECTIVE AND IS CERTAINLY
RELATIVELY EXPENSIVE AND EVEN
THOUGH IT GETS TOP BILLING
BECAUSE IT HAS BEEN AROUND A
LONG TIME, THEY FELT VERY
STRONGLY WE PUT LANGUAGE IN
PRACTICE GUIDELINES ABOUT
POTENTIAL FOR SIDE-EFFECTS TO
FACILITATE MEANINGFUL AND SHARED
DIALOGUE BETWEEN CLINICIAN AND
PATIENT AND FAMILY AND WHAT
OFTEN HAPPENS IS PATIENTS THAT
DON'T LIKE THESE DRUGS THEY ARE
NEW OR FUNCTIONAL SIDE-EFFECTS
THEY DON'T TAKE THEM AND WE ARE
NOT OPERATING IN REALITY AND
CLINICIAN DOESN'T KNOW WHY WE
ARE NOT GETTING BETTER AND
FAMILY DOESN'T KNOW PATIENT IS
NOT TAKING THE DRUG AND
CAPTURING IN CLINICAL PRACTICE
GUIDELINES GOAL IS LET'S SURFACE
WHAT END RESULTS MIGHT BE TO
TALK ABOUT THEM AND COME UP WITH
PRACTICAL SOLUTIONS.
LET'S GO TO THE NEXT SLIDE.
NEXT ONE.
I WON'T SPEND TIME HERE AND
SAYING THAT ENGAGEMENT SHOULD
HAPPEN BEFORE AND DURING AND
AFTER AND ALL THE WAY THROUGH
THE RESEARCH PROCESS AND THERE
IS NOTHING TOO EARLY BUT NOTHING
TOO LATE AND UP FRONT CONSTRUCT
AND CONCEPT.
LET'S GO TO THE NEXT SLIDE AND
GOING TO THE NEXT ONE, SORRY IF
I MAKE YOU CAR SICK WITH THE
CHANGES.
LET'S STOP HERE. TWO CUE THINGS
AND HOW TO DO THIS WELL AND
TRAINING AND ONBOARDING HAS TO
BE FOR EVERYBODY AND RESEARCH
AND SCIENCE BASICS FOR BASICS
PARTNERS GOAL IS NOT TO TURN
NOOSE RESEARCHERS AND LIVED
EXPERIENCE IS EXPERTISE AND IT
MAKES GOOD SENSE TO GIVE US
BASIC AND FOUNDATIONAL TERMS TO
REALLY BE MEANINGFULLY INVOLVED
IN CONVERSATIONS AND DECISION
MAKING AND THINGS LIKE WHAT ARE
QUALITATIVE METHODS OR
QUANTITATIVE METHODS AND WHAT IT
MEANS FOR A STUDY TO BE
WELL-POWERED AND WHAT IS
CLINICAL AND STATISTICAL
SIGNIFICANCE?
THIS ALLOWS US TO BE CONVER SENT
AND TRULY MEANINGFUL PARTNERS
AND CRITICAL TYPE OF ONBOARDING
NOTE IS ENGAGEMENT OF ONBOARDING
THAT IS FOR EVERYBODY.
THIS MIGHT BE COMPLETELY NEW FOR
CLINICIANS AND RESEARCHERS AND
PATIENT PARTNERS AND JUST
UNDERSTANDING HOW WE WORK
TOGETHER AND HOW WE HAVE THESE
MAY BE DIFFICULT CONVERSATIONS
SOMETIMES IF WE HAVE DIFFERENT
OPINIONS AND THOUGHTS AND GREAT
NEWS IS YOU DON'T HAVE TO INVENT
ANY OF THIS.
THERE IS MORE AVAILABLES THAN
YOU COULD EVER USE AND GO TO
PECK ORRI WEBSITE TO FIND THERE
AND WE HAVE DEVELOPED ENTIRE
MODULES AND TRAINING ENTIRE
RESEARCH TEAMS THAT IS ALL
AVAMABLE. YOU HAVE TO MAKE TIME
AND SPACE TO USE IT AND OTHER
PIECE THAT IS NOT TALKED ABOUT
AS MUCH IS NEED TO FACILITATE
ENGAGEMENT IT IS NOT I LIKE TO
SAY IT IS NOT LIKE KOOL-AID YOU
CAN'T JUST ADD WATER OR PATIENCE
OR MIX THEM IN. THERE HAS TO BE
THOUGHT AND BE VERY INTENTIONAL
AND MEANS PREPARATION BEFORE
ACTIVITIES AND MEETINGS DURING
ACTIVITIES AND MEETINGS AND
FOLLOW UP AFTER AND GOING TO
NEXT SLIDE I WILL SHARE NICE AND
TANGIBLE EXAMPLES WHAT THIS
LOOKS LIKE AND SAY YOU HAVE
PATIENT PARTNERS ON RESEARCH
TEAM AND MAYBE LOOKING AT
CURRENT ENROLLMENT NUMBERS FOR
PROJECT.
RATHER THAN SHARING DATA AND
SAYING WHAT DO YOU THINK,
PROVIDING MORE GUIDANCE AND
ALMOST QUEUING SO PEOPLE KIND OF
KNOW WHAT ARE -- WHAT DO YOU
WANT ME TO REACT TO AND WHAT IS
MY JOB HERE?
QUESTIONS LIKE WHAT GAPS DO YOU
SEE?
DO YOU HAVE IDEAS WHY THESE GAPS
EXIST?
WHAT COULD WE DO TO BETTER FILL
THE GAPS?
THINK ABOUT RECRUITMENT
STRATEGIES.
MAYBE IS SHARING INCOMING DATA
AND YOU ARE MAYBE IN HEART OF
STUDY LOOKING FOR THEMES AND
FINDINGS AND STARTING TO DRAW
CONCLUSIONS AND TALKING TO
PATIENT PARTNERS ABOUT IS THIS
CONSISTENT WITH WHAT YOU WOULD
EXPECT AND WHAT YOU SEE IN YOUR
COMMUNITY?
IS ANYTHING SURPRISING?
DO YOU HAVE QUESTIONS OR DO WE
NEED TO DO FURTHER SUBANALYSIS
WHAT WE ARE SEEING HERE?
REALLY PULLING EVERYBODY INTO
THE CONVERSATION.
SO THAT IT IS MEANINGFUL AND
REALLY AUTHENTIC.
GOING TO NEXT SLIDE, THIS CAN
EVEN HAPPEN WHEN IT IS HIGHLY,
HIGHLY TECHNICAL DISCUSSIONS AND
ONE EXAMPLE THAT I DEALT WITH
RECENTLY IS WE NEEDED INPUT ON
DIFFERENT DRUGS AND CLINICIANS
AND RESEARCH WAS FOCUSED WAS ON
MECHANISM OF ACTION AND WHAT IS
TARGET OF THE DRUG?
FELT PRETTY FAR AFIELD FROM
WHERE PATIENTS WOULD BE FOCUSED.
IF YOU CROSSWALK ISSUES TO
THINGS THAT TOUCH PATIENT WLZ IT
IS PHYSICALLY TOUCHING THEM OR
EMOTIONALLY TOUCHING OR
FUNCTIONALLY OR PRACTICALLY,
MAYBE IT IS A DRUG A IS
INJECTABLE AND DRUG B IS ORAL OR
MAYBE DRUG A HAS THIS SIDE
EFFECT PROFILE AND DRUG B HAS
THIS SIDE EFFECT PROFILE.
THOSE THINGS WILL CERTAINLY DRAW
INPUT AND -- AND, YOU KNOW,
OPINIONS AND THOUGHTS FROM
PATIENT PARTNERS AND TAKES A
LITTLE BIT OF MENTAL MATH AND
THINKING THROUGH WHERE DO THESE
TOUCH THE PATIENTS AND WHERE WE
CAN KIND OF GROUND CONVERSATION
AND WILL CLOSE IF WE CAN GO TO
NEXT SLIDE I WANT TO FLAG IF YOU
HAVEN'T SEEN IT NEWLY DEVELOPED
PECK ORRI GUIDANCE I'M STILL
VERY PROUD AND EXCITED ABOUT
ENGAGEMENT RUBRIC CREATED A LONG
TIME AGO AND 10 YEARS NOW AND
NATURAL GROWTH THEY NOW CREATED
A NEW AND IMPROVED MUCH MORE
FULLY FLESHED OUT GUIDANCE AND
SIX FOUNDATIONAL EXPECTATIONS
FOR PARTNERSHIP AND RESEARCH AND
IF YOU HAVE NOT LOOKED AT THEM,
IT IS AN ENTIRE GUIDANCE ITSELF.
I REALLY RECOMMEND IT AND MY
LAST SLIDE IS A LIST OF
RESOURCES THAT I THINK I'M AT
TIME.
I WILL HAND IT OVER TO MY NEXT
COLLEAGUE.
THANKS, AGAIN, FOR THE
OPPORTUNITY TO BE HERE.
>> THANK YOU. IT IS REALLY
SUPERB.
JUST A GREAT WAY TO START THIS
CONVERSATION.
WE DO HAVE TIME AND PLAN TO HAVE
TIME AT THE END TO DWET INTO
THESE THINGS IN DETAIL AND IS
THERE CLARIFYING QUESTIONS
BEFORE WE MOVE ON TO PETER?
PETER, I HOPE YOU ARE SHOWING
YOUR SLIDES OR HAS SOMETHING
HAPPENED IN THE INTERIM?
OKAY. GOOD. PETER IS -- DR.
AMERICA OE IS INTERNATIONALLY
RECOGNIZED RESEARCH IN CLINICAL
EXPERT IN VAS CALITIS AND AUTO
SYSTEMIC IMMUNE DISEASES
RECEIVING FROM YALE AND
UNIVERSITY OF PENNSYLVANIA AND
FELLOWSHIP AT MASS GENERAL
HARVARD MEDICAL SCHOOL AND
SERVES AS CHIEF OF RHEUMATOLOGY
AND PROFESSOR OF MEDICINE AND
EPIDEMIOLOGY AND RECEIVED MOST
FUNDING FROM NIH AND FROM FDA
PECK ORRI AND OTHER INDUSTRY
SOURCES AND FOUNDATIONS AND
PRINCIPAL INVESTIGATOR HOLDING
INTERNATIONAL RESEARCH
INFRASTRUCTURE FOR VAS CLIETS
AND CLINICAL INVESTIGATION AND
FOCUSES ON CLINICAL TRIALS AND
CONDUCT AND OUTCOME MEASURE
DEVELOPMENT EPIDEMIOLOGY AND
BIOMARKER DISCOVERY AND THANK
YOU, PETER.
>> GREAT. CAN YOU HEAR ME?
>> YES. THANKS.
>> GREAT. SORRY THANK YOU. I
HAVE BEEN -- SORRY I MENTIONED
ACTUALLY IN CLINIC THAT I HAVE
SEEN PATIENTS AND WAS ABLE TO
MAKE THE TIME AND APPRECIATE YOU
ASKING ME TO DO THIS AND
DELIGHTED TO BE WITH PATIENT
PARTNERS PART OF THE SAME
SEMINAR AND IS SAME SEGMENT AND
AREA THAT IS IMPORTANT TO ME AND
THINK SHOULD BE IMPORTANT TO ALL
OF US AND HAS GONE REALLY WELL
FOR US.
LET ME -- HOLD ON. PEOPLE FROM
NIH TALKING TO ME AND WILL TALK
ABOUT ENGAGING PATIENTS AND
CLINICAL RESEARCH FROM
INVESTIGATOR'S EXPERIENCE AND MY
PERSPECTIVE AND WILL YOU HEAR
SAME THINGS YOU HEARD WHICH IS
GOOD AND REASON FOR THAT. WE
HAVE SIMILAR EXPERIENCES MOVING
FORWARD AND I LEND INTERNATIONAL
RESEARCH VRVC AND SUPPORT FROM
NIAMS AND NOT WHAT I WILL REFER
TO AT THIS POINT AND MORE OTHER
NETWORKS THAT IS ALL INTEGRATED
AND LET ME CHANGE AND TODAY I
WANT TO TALK ABOUT IMPORTANCE OF
ENGAGEMENT IN CLINICAL RESEARCH
AND EVOLUTION OF PATIENT
ENGAGEMENT AND VAS CALITIS AND
MAIN AREA OF RESEARCH AND
SIMILAR EVOLUTION AND HAS TAKEN
LONGER.
AND THIS IS FROM OUR -- MY
PERSPECTIVE.
LESSONS LEARNED REGARDING
PATIENT ENGAGEMENT IN CLINICAL
RESEARCH AND EXAMPLES OF
SUCCESSFUL PATIENT ENGAGEMENT
TAKING QUESTIONS AS WE MAY DO AS
A GROUP AFTERWARDS AND THINK
THAT THE IMPORTANCE AND BENEFITS
OF PATIENT ENGAGEMENT IN
CLINICAL RESEARCH HAS REALLY
INCREASED RECOGNITION IN PAST 10
TO 20 YEARS OF IMPORTANCE AND
INCLUDING PATIENTS AND RESEARCH
ENTERPRISE AND ACCELERATED TO
POINT THAT ROUTINE SHOULD BE IN
MANY WAYS AND PATIENT ENGAGEMENT
IS MUCH MORE THAN WHAT WE WOULD
HAVE SAID IN THE PAST THAT IS
BETA TESTING FORMS RECRUITMENT
HEALTH GETTING LETTER SUPPORT
FROM PATIENT ADVOCACY GROUP AND
ARE IMPORTANT AND FORM OF
ENGAGING AND IS NOT DIRECT
ENGAGEMENT IN THE RESEARCH
PROCESS.
I WILL REFER TO PATIENT RESEARCH
PARTNERS, PRPS AND LOTS OF
DIFFERENT THINGS DIRECTLY
INVOLVED IN RESEARCH
PRIORITIZATION AND STUDY DESIGN
AND PROJECT LIMITATION AND DATA
ANALYSIS AND DISSEMINATION OF
RESEARCH AND OVER TIME AND THAT
IS THE SORT OF FULL DECK MODEL
THAT I THINK WE HAVE BEEN ABLE
TO SUCCESSFULLY DO AND HAS
WORKED WELL.
BEST OF CLINICAL RESEARCH
CONSORTIUM INTERNATIONAL
RESEARCH WE DO TRIALS TRAINING
BIOMARKER AND WE HAVE A
REPOSITORY AND ALSO FORM
SOMETHING CALLED [INDISCERNIBLE]
WE ARE DOING PATIENT ENGAGEMENT
BEFORE AND HAVE REALLY STEPPED
IT UP AND THINK THAT THIS IS THE
BOTTOM LINE AS ENGAGING PATIENTS
AND RESEARCH IMPROVES RESEARCH
AND MIGHT BE MORE IMPORTANT AND
IMPACTFUL AND STUDY OF RARE
DISEASE BUT NOT SURE I ONLY
STUDY RARE DISEASE.
EVOLUTION OF THINKING ABOUT THE
IMPLEMENTATION OF DIRECT PATIENT
ENGAGEMENT AND RESEARCH?
AGAIN, IT ONLY HAS TO DO WITH
YEARS I HAVE STUDIED AND THERE
IS ALMOST NO ENGAGEMENT 20 YEARS
AGO AND HAVE SEEN THAT INCREASE
AND PEK ORRI AS MENTIONED IS A
MAJOR PUSH AND PECK CORI
REQUIRES PATIENT ENGAGEMENT
BEFORE IT WAS DEFINED BY PECK
CORI AND BEFORE ANY OF US KNEW
WHAT THAT MEANT.
I THINK THAT WE HAD TO FIGURE IT
OUT WITH PECK ORRI AND
PRIVILEGED TO GET FIRST ROUND OF
PILOTED AWARDS PECK ORRI GAVE
AND GRANT OF AWARD DEVELOPING
PATIENT OUTCOME THAT HAS TO HAVE
PATIENT PARTNERSHIP AND WORKING
WITH PECK CORI WE BEGIN TO THINK
ABOUT PATIENT ENGAGEMENT NEEDS
AND FUNDING FOR PATIENT POWERED
RESEARCH THAT I WILL TALK ABOUT
IN A MOMENT. WE ARE NOT ONLY
ONES THEIR PATIENT ENGAGEMENT IS
INHERENT IN WHAT WE ARE DOG AND
WE WERE THERE IN BEGINNING OF
PECK ORRI WHEN PATIENT
ENGAGEMENT WAS DEFINED AND
WORKED ON AND MANY MORE PROJECTS
IN DRC AND TELL YOU RESEARCH
NETWORK THAT IT IS REQUIRED AND
IS INHERENTLY WHAT WE DO AND I'M
HAPPY TO SAY WE ARE SEEING
PATIENT ENGAGEMENT AND INDUSTRY
WORK AND DO WORK WITH INDUSTRY
AND DEVELOPING NEW DRUGS AS WELL
AND SHOWING QUICK EXAMPLE OF
THAT AND THERE HAVE BEEN
LESSONED LEARNED. YOU DON'T
JUST FLIP A SWITCH.
EVOLUTIONARY PROCESS AND FROM MY
PERSPECTIVE AND THOSE THAT
PATIENTS HAVE WORKED WITH AND IS
ABOUT STUDYING REASONABLE
EXPECTATIONS AND WHAT LEVEL OF
ENGAGEMENT THERE IS THAT PEOPLE
ARE EXPECTING PATIENT PARTNERS
HAS TO BE AND SAME FOR
INVESTIGATORS, BY THE WAY. WHAT
DOES IT MEAN?
DIFFERENT IN DIFFERENT STAGES
AND BASED WHO PATIENTS ARE AND
WHAT PROJECT IS AND SPEED OF
RESEARCH OFTEN SURPRISES
PATIENTS AND IS THE SLOW PACE OF
RESEARCH AND ESPECIALLY CLINICAL
TRIALS AND EVERYBODY WANTS
DISEASE STUDIED AND CURED
YESTERDAY. I DO TOO.
THERE NEEDS TO BE EDUCATION
ABOUT WHY THINGS TAKE AS LONG AS
THEY DO.
WE NEED TO CLARIFY RULES AND I
LIKE TO SAY THAT WE ARE ALL
EQUAL BUT NOT EQUIVALENT AND WE
HAVE DIFFERENT SKILLS AND
THINGS.
TO THINK THAT PATIENTS WILL DO
WHAT THE INVESTIGATORS DO AND
THAT THEY DO AND PATIENTS DO AND
LOSES FLEXIBILITY AND LOSE
RICHNESS OF HAVING DIFFERENT
PEOPLE AND VOICES INVOLVED AND
ONCE WE DO THAT MAKES FOR BETTER
RELATIONSHIP AND LEARN TO USE
EACH OTHER'S RESOURCES.
THIS REQUIRES EDUCATION AND
SHOULD HAVE A GRID HERE OF A 2
BY 2 AND INVESTIGATORS TO
INVESTIGATORS AND TELL YOU WHEN
I INVOLVE INVESTIGATORS LEADING
PROJECT AND INSIST WHEN YOU HAVE
PATIENT PARTNER OR 2 THEY WANT
TO KNOW WHAT IT MEANS AND WE ARE
EDUCATING THEM ABOUT VALUE OF
THAT AND HOW IT WORKS AND
PATIENTS NEED TO -- CHOOSING
RESEARCH AND SITUATION TO HELP
PICK WHICH PATIENTS AND EDUCATE
PATIENT AND PATIENT
INVESTIGATORS BACK AND FORTH AND
TRIAL THAT WILL LAST FOR 5 OR SO
YEARS OR MORE AND IS IT A
ONE-TIME SURVEY OR DIFFERENT
PROJECTS REQUIRE DIFFERENT
LEVELS OF ENGAGEMENT AND CULTURE
OF RESEARCH GROUP IS DIFFERENT
IF DOING FOR A WHILE OR NOT AND
ONE SIZE DOESN'T FIT ALL AND DOG
EVERYTHING OR NOTHING IS NOT
PRODUCTIVE AND IS DIFFERENT WAYS
OF DOING THINGS.
TAKES TIME TO GET THIS RIGHT AND
IS WHAT IS IMPORTANT TO REALIZE.
WHAT ARE ROLES?
ROLES ARE GOING TO LOOK FAMILIAR
TO THOSE THAT ARE INVESTIGATORS
AND PATIENT RESEARCH PARTNERS
FRAFRNG AND ARE NON-EXCLUSIVE
INVESTIGATORS ADVISORS
CO-AUTHORS AND EVERY STAGE OF
DEVELOPMENT AS YOU HEARD I AGREE
THERE IS A ROLE FOR PATIENTS AT
EVERY STAGE CONCEPT DESIGN
PROTOCOL DEVELOPMENT
IMPLEMENTATION RECRUITMENT
ANALYSIS REPORTING DISSEMINATION
OR RESULTS AND MAKES MOST SENSE
FOR CLINICAL TRIAL AND COULD BE
ANY TYPE OF OBSERVATIONAL DATA
ANALYSIS AND ALL SORTS OF
STUDIES.
WE DID RESEARCH HOW WE ARE DOG
PATIENT INVOLVEMENT IN RESEARCH
AND MEDICAL DEVELOPMENT FROM A
COUPLE YEARS AGO IT WAS
QUALITATIVE RESEARCH BASED ON 22
REVIEWS PATIENT PARTNERS STAFF
INVESTIGATORS AND 42 MEETINGS
AND RECORDED MEETINGS WHERE WE
HAD PATIENT PARTNERS ON AND
INVESTIGATORS AND WERE STUDIED
IN COMPREHENSIVE FASHION AND
LEARNED AND ARTICLE GOES INTO
GREATER DEPTH AND IF I LEAVE YOU
WITH NOTHING ELSE TODAY THIS
CONCLUSION PROBABLY SAYS IT ALL
THAT DIRECT ENGAGEMENT IN
RESEARCH DESIGN AND DEVELOPMENT
BY PATIENT PARTNERS IN
CO-LEARNING BETWEEN
INVESTIGATORS AND PATIENT
PARTNERS CAN RESULT IN POSITIVE
AND PRODUCTIVE WORKING
RELATIONSHIP FOR MEMBERS OF THE
MEDICAL TEAM AND BIDIRECTIONAL
ENGAGEMENT DIRECTLY BENEFITS AND
IMPACTS RESEARCH DESIGN
PARTICIPANT RECRUITMENT TO
STUDIES AND SUBJECT TO RETENTION
AND SAYS WHERE IT IS AT AND WE
LEARNED HOW TO DO IT WITH ONE
ANOTHER AND ADD THAT YOU WILL
SEE THAT FIVE OF THESE AUTHORS
ARE EITHER PATIENTS OR PATIENT
ADVOCATES IN PATIENT RESEARCH
GROUP AND WANT TO PARTICULARLY
CALL OUT CELESTE WHO DIED A FEW
YEARS FRAG KIDNEY DISEASE
LONG-STANDING CHAMPION OF
PATIENT ADVOCACY AND ENGAGEMENT
AND EXCELLENT PARTNER TO HAVE
AND DRIVING FORCE IN THIS
MOVEMENT AND FEW EXAMPLES FROM
OUR WORK DEVELOPMENT OF THIS
CALLED [INDISCERNIBLE] THAT IS
ONE OF OUR DISEASES WE STUDY A
LOT. PRO AND DISEASE-SPECIFIC.
THIS IS HARD TO DO AND IS HARD
TO DO IT RIGHT AND HARD TO
DEVELOP A VALIDATED MEASURE THAT
IS PATIENT REPORTED AND IS A
COMPLEX MULTI-YEAR AND
MULTISTAGE PROJECT DOING ACROSS
THREE COUNTRIES AND TWO
CONTINENTS REQUIRING PATIENTS
AND INVESTIGATORS AND DATA AND
LOTS HAS TO HAPPEN AND PATIENT
PARTNERS FROM DAY 1 INVOLVED IN
MEETINGS HELPING WITH ANALYSIS
AND THINKING ABOUT HOW TO DO
THINGS THAT WERE ESSENTIAL IN
WORK WE DID AND WEREN'T DATA
POINTS BUT INVESTIGATORS WITH
US.
WE RESULTED IN FIRST VALIDATED
DISEASE SPECIFIC PRO AND VAS
CLIETSDZ THAT IS BEING
IMPLEMENTED IN CLINICAL TRIALS
AND PAPER PUBLISHED A FEW YEARS
AGO AND OTHER PAPERS WITH IT AND
SERIES OF PATIENT PARTNERS THAT
WERE PART OF THE WORK WE DID
THAT WAS ESSENTIAL AND PATIENT
POWER REED SEARCH NETWORK VPPRN
IS A COMMON -- IT IS FORMED BY
VAS CLIETS CLINICAL RESEARCH
CONSORTIUM AND VAS CLIETS
FOUNDATION THAT CAME TOGETHER
STIMULATE BID PECK CORI CALL FOR
PROPOSALS AND WE HAD A
PARTNERSHIP BETWEEN TWO
ORGANIZATIONS THAT IS RESEARCH
ARM VAS CLIETS FOUNDATION THAT
IS WORLD'S LEADING PATIENT
ADVOES ASSCY GROUP IN VAS CLIETS
AND VERY GOOD SYMBIOTIC
RELATIONSHIP AND SEE ALREADY
FROM THE BEGINNING THERE WAS
PATIENT ENGAGEMENT WHO ARE WE?
PATIENTS AND RESEARCHERS
CLINICIANS ADVOCATES AND FAMILY
MEMBERS AND WORK TOGETHER TO
IMPROVE LIVES OF PATIENTS WITH
VAS CLIETS THROUGH ONLINE
CLINICAL RESEARCH AND I'M CO-PI
WITH JENNIFER GARDEN THIS IS NOT
FIRST PI WE HAD WE HAD DIFFERENT
PATIENT PARTNERS AND WILL TELL
YOU EACH HAS BEEN FANTASTIC. IT
HELPS SHE IS A BIT OF A RINGER
AND PHD LEVEL NEUROSCIENTIST AND
R01S IN PAST AND COMPANY AND
GENE THERAPY AND BIT OF A RINGER
AND REMINDS ME TO REMIND
EVERYBODY THAT OUR PATIENTS HAVE
TREMENDOUS TALENT.
THERE IS -- WE ARE ALL PATIENTS.
THERE IS GREAT TALIENT OUT
THERE THAT CAN BRING TO -- FROM
A PATIENT PERSPECTIVE AND
DECISIONS TOGETHER AND FISCAL
DECISIONS FROM FOUNDATION AND
CHRISTINE YOUNG IS MANAGER AND
WE HAVE A STAFF THAT IS
RELATIVELY TO LEAN OPERATION AND
WE STRIVE TO FIND ANSWERS TO
QUESTIONS FOR PATIENTS
PATIENT-CENTERED AND SUCCESSFUL
WE STUDY EVERY TYPE OF VAS
CLIETS AND 5,000 PATIENTS
ENROLLED ONLINE AND THEY
PROVIDED THIS IS NOT ANONYMOUS
BUT THEY ARE TELLING US WHO THEY
ARE AND ARE AVAILABLE FOR
RESEARCH PROJECTS CONTRIBUTING
DATA REGULARLY AND ANALYZED
ACTIVE COMMUNITY OF PEOPLE
RAISING I WOULD LIKE TO BE PART
OF RESEARCH AND INVOLVES EVERY
TYPE OF VAS CLIETS THIS IS A
LIST NOT UP TO DATE WE CHANGE
ALL THE TIME AND INVESTIGATORS
ON LEFT SIDE YOU WILL SEE JUNIOR
INVESTIGATORS I'M PROUD OF AND
PROJECTS AND RIGHT IS STAFF AND
IN MIDDLE YOU WILL SEE THESE ARE
NAMES OF PEOPLE THAT ARE PATIENT
RESEARCH PARTNERS ON VARIOUS
PROJECTS NO THE JUST SAME PEOPLE
WE PUT OUT A CALL FOR EACH
PROJECT AND HAVE A COMPLICATED
SYSTEM THAT PATIENTS HELP US
SELECT WHO PATIENTS WILL BE FOR
PARTNERSHIPS AND HAS BEEN
SUCCESSFUL.
OUR PROJECTS THEY HAVE TO BE
SOMETHING IMPORTANT TO PATIENTS
THEY SAID IS IMPORTANT AND
PATIENT ENGAGEMENT AND USING
RESOURCES AND WHAT HAVE WE DONE?
HAVE 32 PROJECTS 11 COMPLETED
ONGOING ONES AND HAD OVER 1
HUNDRED INVESTIGATORS AND
PATIENT PARTNERS 27 PAPERS
IMPORTANT METRIC AND MULTIPLE
GRANTS HAVE BEEN AWARDED TO
JUNIOR AND SENIOR INVESTIGATORS
AND OTHERS.
EVERY PAPER NEEDS REQUIREMENTS
AND GIVING EXAMPLE THIS IS AERMY
COUPLE YEARS AGO STUDY WEEKS AND
MONTHS AND YEARS FOR BAD
DISEASES TOCH 3 TOP SURVEY
PATIENTS WHAT YOU WANT US TO
STUDY AND DO WORK ON THIS AND
HIGHLY CITED REPORT THAT WE
PUBLISHED SEVERAL YEARS AGO
INTERESTINGLY THIS REPORT AND
PATIENT PARTNERS IN THERE GOT US
THINKING PRELIMINARY DATA LED US
TO WORK WITH OTHERS AND DO
PREDICTIVE ANALYTICS AND BEST
ACRONYM EVER AND PATIENT
RESEARCH AND PARTNERS ENGAGED
AND WOULDN'T HAVE HAPPENED
WITHOUT FIRST STUDY THAT
INSPIRED THIS.
CRITERIA FOR RESPONSE TO
TREATMENT AND VAS CLIETS AND ACR
IS MAJOR PROFESSIONAL GROUP IN
UNITED STATES AND EUROPE AND
AREA OF IMPORTANCE AND
DEVELOPING OUTCOME MEASURE AND
THOSE KNOW THEY ARE IMPORTANT TO
HAVE AND HARD TO DO AND NEARLY
DONE AND PROJECT PATIENT
PARTNERS DAY 1 AND DIRECTLY
INVOLVED IN WORK WE DO AND US
SIDE AND EUROPEAN SIDE AND
EXPERT PANEL THAT IS IMPORTANT
AND ADDING PATIENTS TO THIS VERY
IMPORTANT AND MULTIPLE COUNTRIES
INVOLVED IN THIS AND WILL SHOW
YOU WHY I'M SHOWING YOU TRIAL I
PUBLISHED A COUPLE MONTHS AGO.
IMPORTANT STUDY AND SPONSORED BY
ASTO ZENNICA HAVING A SECOND
DRUG AND LOTS OF INFORMATION AND
PROUD OF THE STUDY AND WHAT WE
DID IN THE MIDDLE IS DID
QUALITATIVE RESEARCH WITH
PATIENTS ADDING ANCILLARY STUDY
UNDERSTANDING PATIENTS
PERSPECTIVES ON TRIAL OUTCOMES
FEELING ABOUT BEING PART OF
TRIAL AND HOW THEY FEEL ABOUT
OUTCOMES AND PATIENT RESEARCH
AND PARTNERS PART OF STUDY TEAM
AND VPR NCN VOLUNTEERED US
SELECTING PATIENTS TO BE
INVOLVED WITH ASTO ZEN EBBINGA
TEAM AND THOSE INVOLVED IN THIS
AND SEE VARIETY OF WAYS PATIENTS
ARE INVOLVED IN OUR WORK AND
MANY WAYS THAT THEY CAN BE
DIRECTLY ENGAGED IN RESEARCH AND
DOESN'T HAVE TO BE ALL OR
NOTHING BUT COULD BE DIFFERENT
THINGS AND PEOPLE HAVE TO WARM
UP TO IT AND DEVELOP CULTURE AND
PRACTICE AND TAKES TIME TO GET
IT RIGHT ENGAGING PATIENTS WITH
LIVED EXPERIENCE AND DISEASE AND
UNDERSTUDY RESEARCH PARTNERS
DOES MANY THINGS FEASIBLE
IMPROVING QUALITY OF RESEARCH
AND ENJOYABLE AND GREAT TO WORK
WITH PATIENTS MAKING RESEARCH
BETTER AND WILL END THERE.
>> THANK YOU, PETER. I THINK IN
TERMS OF HEALTH OF THE COUNTRY
YOUR COMMENTS ARE WORTH YOUR
DIVERSION FROM CLINIC TO JOIN
US. THANK YOU.
ANY QUESTIONS? ANY HANDS?
DON'T SEE ANY.
>> THAT WAS CLEAR.
>> GREAT. AMY, NOT FAR FROM
THESE SHE IS AN INTERNATIONALLY
RECOGNIZED PATIENT ADVOCATOR
LEADER SPEAKER AUTHOR EDUCATOR
AND LEADER IN PATIENT PARTNER
RESEARCH AND ENGAGEMENT EARNING
BA IN UNIVERSITY OF CALIFORNIA
AND MBA FROM PERDUE AND FORMER
COUNCIL MEMBER AND IS NOW THE
PRESIDENT AND CEO OF HEALTHY
MOTIVATION AND EDUCATION AND
ADVOCACY CONSULTING FIRM IN
SANTA BARBARA AND DIAGNOSED WITH
RHEUMATOID ARTHRITIS AT 18 AND
HAD OSTEO POROSIS AND SPENT 5
YEARS IN WHEELCHAIR AND
UNDERWENT 22 SURGERIES WITH
JOINT REPLACEMENTS AFTER THAT
DEVELOPED AS A RUMENT DEVELOPED
AMERICA'S LARGEST NETWORK OF
YOUNG ADULT ARTHRITIS EDUCATION
AND ADVOCACY PROGRAMS STARTED A
CONSULTING FIRM HEALTHY
MOTIVATION THAT HAS BECOME
RESPECTED MEDICAL AND
MOTIVATIONAL SPEAK ER ADVOCATE
ADVISOR FACILITATOR HUMAN
RESOURCE STRATEGIES ADVISING FDA
CRITICAL PATH PROGRAM PECK CORI
AGENCY HEALTH RESEARCH AND
QUALITY AND ENJOYING DECADE
OUTCOME MEASURES RHEUMATOLOGY
AND NIH DIRECTOR'S COUNCIL OF
PATIENT REPRESENTATIVE AND AMONG
HONORS WAS AWARDED PRESIDENT
SERVICE AWARD BY PRESIDENT
GEORGE BUSH AMERICA'S HIGHEST
HONOR FOR COMMUNITY SERVICE AND
KEY OPINION LEADER MUSCULOCARE
REPAIR PATIENT ENGAGEMENT
RESEARCH CARE AND PATIENT
ADVOCACY IN COMMUNICATIONS AND
WE HAVE BEEN AT THIS TOGETHER
FOR A LONG TIME AND LOOK FORWARD
TO HEARING WHAT YOU HAVE TO SAY
TODAY. THANK YOU.
>> I DON'T HEAR ANY.
>> YOU ARE MUTED, AMY.
>> >> HOY ABOUT NOW.
>> GOT IT.
>> GREAT. THANK SO YOU MUCH,
BOB. WE GO A LONG WAY AND I WAS
IN THE CLASS OF 99 THAT IS
GRADUATION CLASS OF NIAMS
ADVISORY COUNCIL AND MUST SAY TO
CREDIT OF DR. CRISWELL I RECALL
MANY TIMES DURING ADVISORY
COUNCIL MEETINGS EVER SO LONG
AGO THAT CONCEPTS OF BUDGETS
WERE LESS THAN 10 MINUTES IN
CONVERSATION OUT OF A DAY-LONG
MEETING AND HAVING THAT LEVEL OF
DETAIL AS AN IMPORTANT SUBJECT
MATTER FOR YOU ALL TO DISCUSS IS
EXTREMELY IMPORTANT.
I CREDIT YOUR LEADERSHIP FOR
THAT AND 27 YEARS LATER I'M
HARDENED BY TYPES OF INITIATIVES
BRANDED WITH VARIETY OF
DIFFERENT NAMES THAT HAVEN'T
CHANGED AND WE DON'T UNDERSTAND
THEM AND REMEMBER WHAT THEY
STAND FOR BUT WHAT THEY ARE DOG
IS SO GOOD AND THANK YOU FOR
YOUR LEADERSHIP IT IS A LEISURE
TO BE HERE TO SUPPORT TWO GREAT
COLLEAGUES AND PETER I HAVE
KNOWN YOU FOR A LONG TIME
WORKING AT INTERNATIONAL LEVEL
AND WE GO BACK SUCH A LONG WAY
IT IS GOOD TO SEE IN SOME WAYS
DOING WHAT I USED TO DO AND
CALLING HER MY LITTLE SISTER IN
PATIENT ENGAGEMENT HAS DONE A
GREAT JOB WUS AND EXPOSURE WE
NEED TO TALK ABOUT PE PATIENT
ENGAGEMENT AT A SYSTEMIC LECHL.
I KNOW THAT CONCERN AND INTEREST
IS WITHIN NIAMS THAT IS WHERE MY
ATTENTION IS GOING TO BE DRAWN
AND BOB ASKED ME TO LOOK AND
TALK ABOUT WHAT IS GOING ON IN
THE FIELD AND PATIENT ENGAGEMENT
AND HOW IMPORTANT IT IS WHO IS
DOING IT BETTER OR WORSE AND WHO
TRIED WHAT TALKING ABOUT THAT
AND EXPERIENCE I WILL SHARE WITH
YOU TODAY IS INFORMED BY ALL OF
THESE ORGANIZATIONS I HAVE BEEN
ASKED TO PARTICIPATE IN AND
COMPETED TO PARTICIPATE IN
VOLUNTEERED TO PAFRPT IN
INTERNATIONAL LECHLS TO UN DOWN
TO LOCAL LEVEL AND OPPORTUNITY
FOR PATIENT LIKE ME WITH
MULTIPLE LEVELS AND I COULD NOT
WALK OR BRING A SPOON TO MY
MOUTH TO EAT THIS IS FROM COMING
FROM A FAMILY WITH NO HISTORY OF
INFLAMMATORY ARTHRITIS
WHATSOEVER AND ASIAN 4TH
GENERATION FAMILY THAT DOESN'T
TALK ABOUT WHAT AIMS THEM.
FOR THE EARLY DAYS OF MY LIFE I
HAD TO TALK ABOUT STAIRS I FELL
DOWN WHEN WEARING A NECK BRACE
NOT BECAUSE I FELL DOWN THE
STAIRS BUT COULDN'T TALK ABOUT A
DISEASE STATE THEY COULDN'T
UNDERSTAND.
NEXT SLIDE, PLEASE -- AS WELL AS
SCHOOL OF HARD KNOCKNOCHL I WENT
THROUGH IN TERMS OF BEING
MEMBERS AND NOTING WHERE I HAVE
BEEN A MEMBER AND CREATION OF
PROCEDURES AND STRATEGIES TO
ENGAGE PATIENTS AND DEVELOP A
SYSTEMATIC WAY TO ENGAGE
PATIENTS AND CURSIORY AT TOP
SUES AND PETER MENTIONED THIS IS
SITUATION FUNDED BY RESEARCH AND
TARGETED YOU KNOW NOT A
REQUIREMENT THAT PATIENTS BE
INVOLVED AND QUESTION BECOMES
HOW I FIND RIGHT PATIENTS AND
FIND PATIENTS WITH EXPERIENCE
AND SCIENCE THAT CAN ARTICULATE
THEIR DISEASE OR EXPERIENCE OF
DISEASE IN WAY TO BE HELPFUL TO
MOVE FORWARD PEK ORRI IS PLACE
THAT AGREE AND PECK ORRI
AMBASSADOR MY JOB IS EXPLAINING
WHAT PEK ORRI IS ABOUT
EXPLAINING WHY WE DO WHAT WE DO
AND HOW IMPORTANT IT IS SUBJECT
MATTER MATTERS TO PATIENTS AND
TOPICS WILL MATTER.
BLOOD AND TESTS AND X-RAY TESTS
MATTER IN SOME WAYS AT CURSORY
LEVEL YES AND DEVELOPMENT STAGES
MAYBE NOT MAYBE THERE ARE
PATIENTS OUT THERE THAT ARE
X-RAY TECHNICIANS AND THERE ARE
ALL DIFFERENT KINDS OF PATIENTS
THAT ARE IMPORTANT TO SIT AT THE
VEMMENT TABLE FROM CONCEPT TO
IMPLEMENTATION TO WRITE UP TO
DISSEMINATION AND ANALYSIS AND
MORE THERE ARE THOSE PATIENTS I
HAVE BEEN AND SUES AN HAS BEEN
PATIENT AND MANY OTHERS AND BULK
OF PATIENT PARTNERS ARE DISEASE
SPECIFIC AND TOPIC SPECIFIC AND
MIGHT BE JOINT REPLACEMENTS AND
THOSE THINGS THAT ARE VERY
SPECIFIC TO THAT PARTICULAR
TOPIC AND FINDING THEM IS A BIG
KEY TALKING ABOUT WHAT IS
HAPPENING AT FDA LEVEL AND
SUBJECT THAT YOU WILL ADDRESS
AFTER PRESENTATIONS AND
DISCUSSIONS AND FDA LEVEL HAS
BEEN STRONG IN THE
PHARMACEUTICAL SIDE AND ALSO THE
CDRH SIDE CENTER FOR RADIATION.
ANYWAY CDRH YOU KNOW WHAT IT
MEANS EXCUSE ME SKIPPED MY MIND
EVEN THOUGH I'M ADVISOR TO THEM.
THEY CAME UP WITH VERY FIRST
PATIENT ENGAGEMENT ADVISORY
COMMITTEE SUSAN AND I HAVE HONOR
SERVING FIRST GENERATION OF THAT
AND AS THIS IS GOING ON WE ARE
ADDRESSING AT THIS POINT OF TIME
STRATEGIC DISCUSSIONS AND
STRATEGY AND HAVE NOT GOTTEN
INTO IMPLEMENTATION AND
ANSWERING QUESTION WHAT
SPECIFICALLY FDA WILL DO AT
SYSTEMS LEVEL AND BEING CONVENOR
OF VARIETY OF SYSTEMS PETER
TALKED ABOUT WELL ESTABLISHED
INTERNATIONAL AND NATIONAL VAS
CLIETS PATIENT POWERED NETWORK
COULD IT BE OVERLAYS IN MULTIPLE
DISEASE TOPICS TALKING ABOUT
NATIONAL TOPICS I WILL TOUCH ON
A COUPLE BEING RESPONSIBLE THE
LAST PETER SAID WE HAVE NOT SEEN
MANY ARTICLES ON PATIENT
ENGAGEMENT BEFORE 20 YEARS AGO.
IF THEY WERE COMING OUT
INFORMATION WAS 1999 TO 2002 AND
PATIENTS INVOLVED EVERY STEP OF
WAY AND RESEARCH AND WRITE-UPS
AND FLAIR TO THEM AND NATIONAL
HEALTH COUNCIL DUG DEEPER AND
DEVELOPED TOOLS TO HELP
RESEARCHERS LIKE YOURSELF BETTER
LOOK AT PATIENTS THAT MIGHT BE A
POTENTIAL PATIENT PARTNER
RESEARCH PATIENT RESEARCH
PARTNER THINGS LIKE HOW TO VALUE
TIME AND WHAT THEY WILL BE DOING
IS THERE TRANSPORTATION
INVOLVED?
THERE IS CHEAT SHEETS I WOULD
CALL THEM FOR YOU TO FILL OUT AS
AN INVESTIGATOR ON THE NATIONAL
HEALTH COUNCIL'S WEBSITE THAT
COULD HELP YOU AND WHAT YOU WILL
DO PRECURSOR HOW YOU WILL
COMPENSATE AND MOVEMENT THERE IS
CHEAT SHEETS TO HELP YOU
DETERMINE WHAT A PATIENT
RESEARCH PARTNER MIGHT BE ABLE
TO DO FOR THE ORGANIZATION.
YOU CAN BUDGET APPROPRIATELY.
FASTER CURES IS AN OTHER
ORGANIZATION AND MICHAEL MILTON
ORGANIZATION AND THEY HAVE
DEVELOPED A FEW WHITE PAPERS
INCREASING DIVERSITY AND
CLINICAL TRIALS STRONG ELEMENT
OF PATIENT ENGAGEMENT AND IN
ADDITION TO THAT THEY COME OUT
VERY SOON AND HAVE BEEN PART OF
THAT VERY SOON WITH ANOTHER
WHITE PAPER, A SEPARATE WHITE
PAPER ON PATIENT ENGAGEMENT AND
TRYING TO GET A SCOPE WHAT IS
GOING ON AND WHERE THE NATIONAL
NEEDS MIGHT BE.
C PAL CENTER FOR PATIENT
ADVOCACY LEADERS I'M A PRINCIPLE
AND THIS IS AN ORGANIZATION OF
MULTIPLE DISEASES MULTIPLE
LEVELS OF PATIENT ENGAGERS ALL
VOLUNTEERS AND/OR STAFF PEOPLE
OF NON-PROFIT ORGANIZATIONS OUR
JOB INFORMING THEM OF LATEST
ISSUES AND PROCESSES AND
ENGAGEMENT HAS BEEN ON TIP OF
TONGUE IN WORK THAT CPALS HAS
DONE AND PARTNERED WITH VARIETY
OF DIFFERENT HEALTH CONSULTANCY
ORGANIZATIONS AND COMMERCIAL
VENTURES TO DEVELOP ACROSS
DISEASE KINDS OF MODELS FOR
THESE GROUPS TO MOVE FORWARD
WITH I MENTION ARTHRITIS
FOUNDATION. I HAVE A HOME THERE
AND BEEN A MEMBER FIRST A
RECIPIENT OF SUPPORT AND PROGRAM
TO SHARE FOR THIS FOUNDATION AND
IN CALIFORNIA AND REPRESENTING
THEM AT A VARIETY OF DIFFERENT
ACTIVITIES AND THAT THE CITIES
IMPORTANTLY IS ABOUT THE
ADVOCACY AND AND TRAINING OF
PATIENTS TO BECOME ADVOCATES AND
TRAINING ADVOCATES TO BECOME
BETTER AND STRONGER ADVOCATES TO
GO BEFORE CONGRESSIONAL HEARINGS
AND NIAMS HEARINGS AND THINGS
LIKE THAT RULE OF NATIONAL
NON-PROFIT PROJECTS IS EXTREMELY
IMPORTANT AND OTHER SIDE IS
INTERNATIONAL I TAKE EFRD-BASED
EXAMPLE 1992 AND TAKING
INTERNATIONAL RESEARCH DEVOTED
TO RHEUMATOLOGY AND OUTCOME
MEASURES TO REALIZE WHO IS
MISSING AT THE TABLE AND WHO IS
MISSING IS PATIENT PARTNERS AND
SLOWLY, AGAIN, NOT
SYSTEMATICALLY BUT SLOWLY
INVITATIONS WERE SENT TO PEOPLE
LIKE ME.
I WAS IN THE FIRST GROUP.
I MUST SAY THAT AT FIRST MEETING
OF PATIENT RESEARCH PARTNERS,
PRPS WAS DONE TOTALLY SEPARATE
FROM OUR RESEARCHER GROUPS THAT
IS 2 HUNDRED OTHER PEOPLE PRETTY
MUCH AND WE WERE MEETING OUTSIDE
UNDER A PALM TREE IN AUSTRALIA
AND YOU KNOW THAT THEY HAD PALM
TREES IN AUSTRALIA AND ALL
INVESTIGATORS WERE MEETING
INSIDE.
THERE WAS SEPARATE AND HAD A
FOCUS GROUP INTERESTING START
AND WE DO NOT USE THAT MODEL
ANYMORE.
PETER HAS ARTICULATED SO WELL
NOW IT IS LIKE VAS CLIETS MODEL
AND MODEL IS FOR ALL PATIENTS
THAT WILL BE ENGAGED AND PATIENT
PARTNER LEADERS WHICH I WAS ONE
HELP THE ORGANIZATION FIND
PATIENTS THAT WOULD BE WILLING
TO SERVE TO BE ON MY ZOOM CALLS
AND TO BE THE RECIPIENT OF
SURVEYS AND FOCUS GROUPS AND DO
IT WILLINGLY AND AS LONG AS WE
CAN UNDERSTAND AND THERE WAS
OVER 10 PEOPLE AND LOOKED AND
SAID HOW CAN WE CONNECT WITH
PEOPLE IN THE ROOM 2 HUNDRED
YARDS FROM US IN A WAY THAT
MAKES SENSE?
WHAT CAME OUT WAS WAIT A MINUTE.
I DON'T UNDERSTAND THEIR
LANGUAGE AND LANGUAGE OF SCIENCE
IS DIFFERENT THAN LANGUAGE OF
[INDISCERNIBLE] AND WE TALK
ABOUT PAIN AND SCIENCE AND
SCIENTISTS TALK ABOUT PAIN IN
MAYBE 2 HUNDRED DIFFERENT WAYS
OF DESCRIBING PAIN AND SO IT
FORCED US TO TAKE A LOOK AT WHAT
IT IS THAT WE NEED TO KNOW TO BE
IN THE SAME ROOM AS OUR
RESEARCHERS AND THAT IS WHERE
THE PATIENTS DECIDED TO CREATE A
GLOSSARY.
WE PRESENTED FINDINGS TO THE
EXECUTIVE BOARD AND THEY AGREED
AND THEY ALLOWED US TO START IT
AND WE HAD YOUNG INVESTIGATORS
ASSIST US.
20-SOME-ODD YEARS LATER GLOSSARY
IS BEING UPDATED ON ANNUAL BASIS
AVAILABLE TO ANY RESEARCHER, NEW
OR OLD AND ANY PATIENT PARTNER
WHO IS INTERESTED IN
RHEUMATOLOGY RESEARCH TO BETTER
UNDERSTAND CONCEPTS AND SCIENCE.
THOSE THINGS THAT PATIENTS TOOK
A LOOK AT AND STARTED TO DO.
URACR CONNECTION AS PETER SAID,
IT IS A REALLY TIGHT
COLLABORATION ACROSS THE
ATLANTIC OCEAN AND THOSE THINGS
THAT WE INITIALLY STARTED OUT
VERY SEPARATE. I WAS FIRST
PATIENT TO BE ASKED TO SERVE ON
A GUIDELINES DEVELOPMENT
COMMITTEE OF ACR.
WE MET IN A ROOM. EVERYBODY
LOOKED AT ME AND KNEW I WAS A
PATIENT.
WENT UP TO OUR CHAIR AND SAID,
WHY IS SHE HERE?
SO THESE ARE SOME OF THE EARLY
COMMENTS THAT IS VERY
INTERESTING.
I CAN TELL YOU THAT 45 MINUTES
LATER DURING THE BREAK AFTER WE
ALL HAD A CHANCE TO SAY OUR
PEACE SO-TO-SPEAK AND WHY WE
WERE THERE AND WHAT WE HOPED TO
ACHIEVE, THREE INDIVIDUALS
RESEARCHERS CAME UP TO ME AND
SAID, NOW I KNOW WHY YOU ARE
HERE. NOW I KNOW WHY YOU ARE
HERE. YOU ARE HERE TO GROUND US
AND BRING US BACK TO REALITY OF
WHAT PATIENTS HAVE TO DEAL WITH
AND ARE DEALING WITH AND WHAT
FAMILIES ARE DEALING WITH AND
ARTICULATE IT WELL AND DOING IT
EFFECTIVELY IN CROWD THAT SPEAKS
DIFFERENT LANGUAGE THAN YOURS
AND SPOKESPERSON INTERNATIONAL
LEVEL FOR [INDISCERNIBLE] AND
NOT EVERYBODY SPEAKS YOUR
LANGUAGE AND DON'T ASSUME THAT
THEY DO SPEAK YOUR LANGUAGE
WHATEVER MEETING THEY ARE AT AND
NO THE THAT WE HAVE TO DOWNSIDE
CONVERSATIONS BUT CERTAINLY LAY
LANGUAGE IS AN IMPORTANT PIECE
AND IS BEST TWO WORDS TO
DESCRIBE BEING REAL AND SPEAKING
TO PATIENTS LIKE YOU ARE
SPEAKING TO NEIGHBOR.
THAT IS A VERY INTERESTING
PERSPECTIVE THAT IS IMPORTANT TO
KNOW THAT YOU ARE ACR AND THAT
OTHER NATIONAL EXPERIENCES IN
LUPUS AND RHEUMATOID ARTHRITIS
AND VAS CLIETS PEOPLE SAID AND
THINK THAT THESE KIND OF
SYSTEMIC DEVELOPMENTS CAN BE
HELP. TO NIAMS AND I WANT TO
LASTLY TALK ABOUT THE COMMERCIAL
SIDE YOU MIGHT SEE IF YOU PUT IN
WORD PATIENT ENGAGEMENT INTO
GOOGLE RESEARCH OR ANY RESEARCH
CERTAINLY NOT ADMIT BUT GENERAL
RESEARCH YOU WILL SEE THAT
PROBABLY 50% OR MORE OF CONTENT
IS BEING DEVELOPED BY FOR PROFIT
HEALTH CONSULTING AGENCIES THAT
SEE A NICHE, IF YOU WILL.
THEY SEE AN OPPORTUNITY BECAUSE
OF VARIETY OF TOPICS THAT WERE
MENTIONED PEOPLE AND PATIENTS
DON'T UNDERSTAND WHAT IT MEANS
AND PEOPLE DON'T UNDERSTAND HOW
TO EMPLOY IT AND THERE IS AN
OPPORTUNITY TO DEVELOP SYSTEMS
AND ONE IN PARTICULAR I WILL
CITE IT IS A CONSULTING FIRM
THAT APPROACHED C HOUSE WITH
INTENT TO DEVELOP FIRST OF ALL A
TASK FORCE AND THEY WANTED TO
GET A HANDLE AND THEY ARE
INTERNATIONAL AND WANTED TO GET
A HANDLE WHAT WAS HAPPENING IN
UNITED STATES AND IN EUROPE AND
WHAT COULD BE DONE AND WHERE ARE
GAPS?
ALSO, WE HAD SUGGESTED AND
PATIENTS SUGGESTED WHY DON'T YOU
KEERY YOUR OWN INDUSTRY AND WHAT
TARGETS ARE YOU INTERESTED IN?
INTERESTED MORE IN MEDICAL
TECHNOLOGY AND MEDICAL DEVICE
FIELD AND ASKED THEM PUTTING
TOGETHER A SURVEY HELPING THEM
DEVELOP THAT AND SURVEY WENT OUT
AND 9 HUNDRED PEOPLE RESPONDED
THAT IS SIGNIFICANT ACROSS A
VARIETY OF DIFFERENT COMPETING
COMPANIES ABOUT PATIENT
ENGAGEMENT THAT PRODUCED FOR THE
TASKFORCE A REAL SHOCK THAT
NEARLY 80% OF THE PEOPLE THAT
RESPONDED HAD NO CLUE ABOUT THE
DEFINITION AND WHAT IT MEANT OR
HOW TO EMPLOY IT.
THEY THOUGHT PATIENT ENGAGEMENT
WAS THEM AND EACH PERSON DOING
THEIR WORK AS INSTRUCTED OR AS
GUIDED BY THEIR BOSS.
IT WAS INTERESTING THAT THEN
WHAT WAS PRODUCED OR VARIETY OF
DIFFERENT DOCUMENTS THAT ARE
USED INTERNALLY AND HAVE A
RESULT OF THAT WILL TELL YOU
THEIR GOAL AND WOULD LIKE TO
DEVELOP A CERTIFICATION PROGRAM
AND EDUCATION PROGRAM FOR MED
TECH STAFF FIRST WITHIN THEIR
COMPANY AND THEN TO GO BROADLY
WITHIN THE MED TECH INDUSTRY AND
CERTIFICATION PROGRAM ON PATIENT
ENGAGEMENT.
AND SO THOSE WHO WERE PATIENTS
IN THE ROOM, ME IN PARTICULAR
SAID THIS IS A SIMILAR SITUATION
WITH PATIENTS.
PATIENTS WANTED TO BE PART OF
THE LOCAL RESEARCH GROUP AND
OTHER PATIENTS WANT AND CAN AND
WISH TO PARTICIPATE AT NATIONAL
OR INTERNATIONAL LEVEL AND HOW
DO WE ENSURE THAT THEY HAVE AT
LEAST KNOWLEDGE THAT CAN BE
GARNERED AND LEXICON THAT WILL
BE USED?
SO THE IDEA OF A CERTIFICATION
PROGRAM FOR STAFF OR MEASURING
PEOPLE IN THE FIELD POSSIBLY FOR
PATIENTS IS SOMETHING TO HOPE
FOR AND NEXT SLIDE, PLEASE.
I WANT TO CONCLUDE WHERE THE
OPPORTUNITIES ARE.
AGAIN, LOTS OF THINGS I TALKED
ABOUT HAVE NOT BEEN WRITTEN UP
YET AND PROBABLY WON'T.
WE ARE SEEING THAT THE CONCEPT
OF IMPLEMENTATION OF PATIENT
ENGAGEMENT IS UNIQUELY DIFFERENT
TO EACH ENTITY AND TO POSSIBLY
EACH THERAPEUTIC AREA AND TO THE
SCOPE OF WHERE YOU ARE GOING TO
DO YOUR WORK GEOGRAPHICAL SCOPE
OR TOPICAL SCOPE.
OPPORTUNITIES FOR NIAMS IS
LEARNING SITUATION FROM ALL
GUIDANCE THAT IS BEING PROVIDED
THAT IN WRITTEN FORM IS PIECE
MEAL BY PIECE MEAL THAT PETER
DEMONSTRATED TO US AND INTENT
AND GLOBAL PIECE THAT SUE HAS
DESCRIBED SO BEAUTIFULLY.
THIS IS A VISION FOR YOU I WOULD
LIKE YOU TO THINK ABOUT.
TODAY I FEEL LIKE A PATIENT
RESEARCH PARTNER AND ENGAGED
PATIENT RESEARCH PARTNER WHEN I
RECEIVE INFORMATION LIKE ANYONE
ELSE ON DEVELOPMENT TEAM IF I AM
ON THE DEVELOPMENT TEAM AND HAVE
THE OPPORTUNITY TO TALK WITHIN
THE GROUP NOT SEPARATELY AND NOT
LIKE A FOCUS GROUP.
BUT TO TALK AND THEN INTERACT.
IF I NEED EDUCATION, THERE IS
ONE OR TWO MDPHDS THAT WILL SIT
DOWN WITH ME AND TALK ABOUT CHI
SQUARES AND DIFFERENT KINDS OF
CONCEPTS THAT WILL BE DISCUSSED
IN ADVANCE SO I KNOW AND CAN
CONTRIBUTE ACCORDINGLY.
THIS DOESN'T HAPPEN THAT OFTEN.
WHEN IT DOES, IT VALUES THAT
NEXT CONVERSATION AND THAT MIXED
INTERACTION IMMENSELY AND
ABSOLUTELY IMMENSELY EVERY STAGE
SUE AND PETER HAVE DISCUSSED
PATIENTS AND RESEARCH PARTNERS
ARE INVOLVED AND BY LOOKING AT
IT AND REVIEWING IT AND INVOLVED
WHY EDITING AND SAYING WHY THIS
DOESN'T MAKE SENSE AND PUTTING
IN EASIER LANGUAGE FOR PEOPLE TO
UNDERSTAND.
OKAY. HOW ABOUT WRITING A LAY
ARTICLE OR LAYMAN'S OR LAY
PERSON'S ARTICLE AS A RESULT OF
THE RESEARCH? AND HAVE THAT GO
OUT TO A WIDER DISTRIBUTION
AUDIENCE?
THAT IS WHERE TO ME IN MY VISION
TRUE PATIENT ENGAGEMENT REALLY
WORKS AND THERE HAS TO BE
TRAINING AND EDUCATION.
THERE HAS TO BE A PROCESS THAT
CAN'T -- HAS TO BE FLUID IN MANY
WAYS.
WE HAVE TO LIKE NIAMS DOES WITH
ITS YOUNG INVESTIGATORS.
WE HAVE TO HAVE A SYSTEM OF
HELPING PATIENTS TO BECOME
BETTER AT TALKING ABOUT THEIR
EXPERIENCE IN A SCIENTIFIC
ENVIRONMENT.
SO THAT IS THE VISION I WOULD
LIKE TO SEE YOU COME UP WITH.
NEXT SLIDE, PLEASE.
I'M STANDING NEXT TO MY MOTHER.
NO. SHE IS NOT MY SISTER. I
HAD THREE PEOPLE ASK ME IN
WASHINGTON, DC IS THIS YOUR
SISTER? THIS IS MY MOTHER WHO
SINCE PASSED AWAY. SHE SHARED
EVERYTHING WITH ME AND TOOK CARE
OF ME WHEN I COULDN'T WALK OR
EAT.
SHE WENT WITH MY ESCORT AND
HELPER AND WORKER AT
INTERNATIONAL LEVEL WHEREVER I
WAS TRAVELING.
AND SHE CAME TO WASHINGTON, DC
AND ADVOCATED AS A CAREGIVER.
WE CAN'T FORGET CAREGIVERS OR
FORGET THIS IS A DISEASE OR SET
OF DISEASES THAT WE WILL LIVE
WITH FOR THE REST OF OUR LIVES
UNTIL YOU CAN COME UP WITH A
CURE.
THANKS SO VERY MUCH. NEXT
SLIDE, PLEASE.
THANK YOU, AMY.
>> YOU JUST LAID OUT THE
JOURNEY. IT HAS BEEN A LONG ONE
BUT AMAZING WHERE WE HAVE GOTTEN
TO.
IF ANYONE HAS ANY GENERAL
QUESTIONS FOR ANY SUE, PETER OR
AMY.
LET ME PUT SOMETHING ON THE
FLOOR.
THAT IS THAT WE SHOULD THINK
ABOUT NIAMS WITH THE ADVICE FROM
COUNCIL HOW TO MAKE SURE WE
INCLUDE ENCOURAGEMENT FOR
PATIENT ENGAGEMENT WHEREVER
APPROPRIATE IN A CLINICAL STUDY.
SO THAT IS WHAT WE WILL TALK
ABOUT HERE.
THIS IS A LITTLE BIT GETTING
AHEAD OF OURSELVES AND THIS
CIRCLES BACK A BIT TO THE
PRESENTATION AND CLINICAL TRIALS
GROUP AND A LITTLE FORESHADOWING
THERE.
IF ANYONE WOULD LIKE TO COMMENT
ON THE QUESTION OF SHOULD WE
WRITE INTO FUNDING OPPORTUNITY
ANNOUNCEMENTS AND WRITE INTO
NOTICE OF REWARD THAT WE EXPECT
PATIENT ENGAGEMENT SOUP TO NUTS
UNLESS OTHERWISE INAPPROPRIATE.
>> LET'S SEE WHAT --
[INDISCERNIBLE] PATIENT ADVOCATE
AND DON'T WORRY. WILL YOU COME
DOWN.
ALSO FROM PEOPLE AND NIAM HAS
BEEN IN THE ROLE AND PEOPLE ALSO
WHO HAVE BEEN ENGAGED IN
RESEARCH AND WE ONLY HAVE 10
MINUTES AND THINK A FEW COMMENTS
WOULD HELP STUDENTS UNDERSTAND
WHERE TO GO WITH THIS.
>> FIRST OF ALL, THANKS FOR
PUTTING THIS ON THE TANL. IT IS
AN IMPORTANT TOPIC AND REALLY
GRATEFUL YOU HAVE BROUGHT THIS
FOR DISCUSSION.
I THINK IT WILL BE CRITICAL TO
INCORPORATE THIS AS A
REQUIREMENT WITHIN THE GRANT
APPLICATIONS FOR CLINICAL
STUDIES THAT IS ONLY WAY IT IS
GOING TO BE DONE PROACTIVELY AS
ALL SPEAKERS HAVE MENTIONED
ENGAGING PATIENTS NEEDS TO BE
INCORPORATED THROUGHOUT PROCESS
AND NOT AS AN AFTERTHOUGHT AND
THINK IF NOT INCLUDED AS PART OF
THE APPLICATION IT IS REALLY
MUCH LESS LIKELY TO BE FRONT AND
CENTER IN ALL EFFORTS AND THERE
IS ACTIVITIES THAT ARE HAPPENING
AND DON'T THINK INVESTIGATORS
NEED TO REINVENT THE WHEEL AND
CAN TAP INTO RESOURCES THAT
EXIST FROM PATIENT ADVOCACY
ORGANIZATIONS AND INVESTIGATORS
LEADING EFFORTS AND LOTS OF
PUBLICATIONS AND ARE LOTS OF
RESOURCES OUT THERE THAT COULD
BE UTILIZED RATHER THAN
SOMETHING INVENTED FROM SCRATCH.
>> I'M CERTAINLY -- THANK YOU
FOR THE PRESENTATION AND AS I
THOUGHT ABOUT IT, I CONCUR WITH
RECOMMENDATION THAT WOULD HAVE
TO BE REQUIRED TO REALLY -- AS
PECK ORRI DOES THAT I'M MOST
FAMILIAR WITH SOME OF THE OTHER
PROGRAMS FUNDED UNDER THERE.
THERE IS A GREAT TRAINING NEED
FOR THINGS NIAMS COULD TAKE THE
LEAD IN GETTING OUT INTO THE
COMMUNITY AND WHAT IT MEANS.
ONE OF THOSE SUGGESTIONS I WOULD
HAVE, HAVING HEARD THESE
EXCELLENT PRESENTATIONS IS IN
SOME WAYS THEY ARE AT A HIGH
LEVEL IN GENERAL.
IT WOULD BE HELPFUL TO HEAR
EXAMPLES OF WAYS THAT DON'T WORK
FOR PATIENT ENGAGEMENT OR
PRETEND TO BE PATIENT ENGAGEMENT
AND HAVE DISASTROUS RESULTS IN
TERMS OF CLINICAL TRIAL RESULTS.
THIS SLIDE THAT STANDS OUT IN MY
MIND THAT IS A RETURN ON
ENGAGEMENT THAT SUE PRESENTED.
I WOULD HAVE LIKED OR WOULD
STILL LIKE TO HEAR MORE CONCRETE
SPECIFIC EXAMPLES AND THINK WHEN
YOU ARE DOING TRAINING IT WOULD
BE VERY HELPFUL TO SAY WHY IT
MAKES SUCH A DIFFERENCE WITH
HANDS ON THE GROUND EXPERIENCE
WHAT WORKS AND WHAT DOESN'T AND
EITHER CLINICAL TRIALS END UP
NOT -- I WON'T GO INTO SPECIFICS
BUT THINK THAT THEY WOULD BE
VERY HELPFUL.
>> YEAH. THANK YOU. FIRST OF
ALL I WANT TO THANK OUR THREE
PRESENTERS FOR THESE FABULOUS
PRESENTATIONS AND MANY OTHER
WAYS WHICH THEY FELT THAT THE
INSTITUTE MOST RECENTLY PETER
AND CLINICAL TRIALS WORKING
GROUP HEAR ABOUT THAT AND SUE
RECENTLY COMPLETED TIME AS A
COUNCIL MEMBER AND AMY, YOU
HELPED US RECENTLY WITH A
REGENERATIVE MEDICINE ROUNDTABLE
REPRESENTING PATIENT PERSPECTIVE
THAT IS SO VALUABLE.
THANK YOU ALL.
AMY I DON'T KNOW IF YOU REMEMBER
WE MET A MILLION YEARS AGO WITH
KICKOFF OF BIG RHEUMATOID
ARTHRITIS GENETICS CONSORTIUM
AND REMEMBER YOU FOR SO LONG AND
ON THIS TOPIC, I AGREE WITH FOR,
YOU KNOW, WE SHOULD BE PROACTIVE
IN THIS SPACE AND ARTICULATE
EXPECTATIONS THAT IS CRITICALLY
IMPORTANT THAT WE MAKE IT AS
EASY AS POSSIBLE FOR
INVESTIGATORS TO DO THIS.
THERE ARE PAPERS AND RESOURCES
AND WHAT CAN WE DO TO REALLY
BRING THIS LITERATURE AND
KNOWLEDGE AND THESE APPROACHES
TO THE RESEARCHERS.
IN FACT, ONE REASON WHY I WAS
EAGER TO HAVE THESE
CONVERSATIONS IS THAT YOU KNOW
I'M HEARING FROM INVESTIGATORS
WE WANT TO DO THIS BUT HOW? I'M
NO THE SURE HOW AND WE WILL DO
WHAT YOU ARE PROPOSING THAT IS
TO IDENTIFY YOU KNOW BEST
PRACTICES RESOURCES AND COMPILE
THEM OR SOMEHOW DISTILL THEM AND
PRESENT THEM TO OUR COMMUNITY
AND TO OUR INVESTIGATORS TO
ACTUALLY DO THIS.
I AGREE ALSO NOT ONLY DOES IT
FEEL LIKE ETHICAL AND MORAL
THING TO DO AND FOR THAT REASON
WE SHOULD DO IT AND ALSO THE
HARD DATA THAT RE-ENFORCES VALUE
IN ALL DIFFERENT WAYS YOU KNOW
COST EFFECTIVENESS IN THE LONG
RUN AND FAILED TRIALS AND YOU
KNOW ET CETERA.
SO I -- THAT LITERATURE SEEMS
LIKE A BIT OF A BLUR TO ME AND
I'M NOT I DON'T HAVE A GOOD
HANDLE ON IT AND WONDER WHETHER
WE NEED AN ONGOING EFFORT OR
SOMETHING ELSE WITHIN THE
INSTITUTE OR IN COLLABORATION
WITH COUNCIL SO THAT YOU KNOW AS
WE START EXPECTING THIS AND
REQUIRING IT OUR INVESTIGATORS
CAN BE PREPARED AS POSSIBLE TO
IMPLEMENT.
GREAT. WE SEE A COUPLE HANDS UP
HERE.
PETER AMERICALE AND CARL YEAH
PETER.
>> THANKS.
>> I'M -- I AGREE THAT THIS
SHOULD BECOME MORE PART OF THE
NORM.
I ALSO AGREE THAT ANNE MADE
INTERESTING POINTS SAYING WHAT
DOESN'T WORK AND SOMETHING FOR
WORK AND FIRST OF ALL NOT SURE
THIS FOR EVERYTHING AND EVERY
PROJECT AND DOING IN ANALYSIS
AND LOOKING AT NEW METHOD MAY
NOT BE NECESSARY TO HAVE
INNOVATION ENGAGEMENT AND MAY BE
AND DEPENDING ON IT AND TRIALS
DEFAULT IS YES AND PENALTY PHASE
AND YOU NEED TO THINK ABOUT IT
AND OTHER THING IS YOU SHOULD DO
IT BECAUSE IT WILL HAVE VALUE,
WHICH I THINK IT DOES.
YOU ALSO HAVE TO REALIZE THAT
LOTS OF THE PATIENTS COULD BE.
THESE ARE TECHNICAL TERMS AND
THINK THAT THE SAME THING CAN
HAPPEN AND I THINK THAT IS WHY
WE WANT TO -- YOU WANT TO PICK
YOUR RESEARCH COLLABORATORS VERY
CAREFULLY FOR THE SAME REASONS
AND YOU ALSO COULD HAVE THE SAME
THING AND DANGEROUS GROUND HERE.
I HAVE BEEN INVOLVED IN LOTS OF
STUDIES AND IF YOU ALWAYS HAVE
THE SAME PEOPLE AND YOU NEED
OTHER VOICES AS WELL. THAT IS
TRUE IN RESEARCH AND ALSO FOR
PATIENT PARTNERS AND THEY OFTEN
HAVE CERTAIN PEOPLE THAT ARE
ALWAYS THERE.
THAT MAY NOT BE A GREAT IDEA
EITHER.
SO, I THINK, YOU KNOW, YOU TALK
ABOUT LESSONS LEARNED AND THINGS
THAT DON'T WORK.
THOSE ARE IMPORTANT.
I THINK WE WANT TO EMPOWER
INVESTIGATORS AND GIVE THEM I
THINK YOU KNOW CARROTS I THINK
WORK BETTER THAN STICKS.
MONEY DOES ACTUALLY IT IS
SHOCKING TO THINK INVESTIGATORS
GO ACTUALLY TOWARDS THE MONEY OR
TOWARDS THE REQUIREMENTS OF RFAS
BUT THEY DO AND THERE NEEDS TO
BE A RAMP UP PERIOD AND PEOPLE
GET SORT OF USED TO IT AND WE
HAVE TO BE WORRIED ABOUT TOKING
PATIENT ENGAGEMENT THAT IS NOT
REAL AND ALSO OVEREXPECTATIONS.
IT IS A BALANCE AND LIKE YOU
KNOW IT WHEN YOU SEE IT.
WE ARE NOT -- I WILL TELL YOU WE
DON'T HAVE PATIENT WITH
EVERYTHING WE DO AND MORESO WE
ARE AND THAT IS THE TYPE OF
THING.
THERE IS NO ONE RULE BUT SOME
SORT OF APPROACH.
I THINK YOU HAVE TO HELP PEOPLE
UNDERSTAND WHAT THIS IS.
BUT I THINK THAT IS TRUE FOR
OTHER THINGS AND ANALOGY IS
BASIC INVESTIGATORS FEEL THEY
KNOW HOW TO DO CLINICAL
INVESTIGATION AND DO IT AND IT
IS USUALLY NOT A GREAT IDEA AND
THEY NEED HELP AND IT IS THE
SAME TYPE OF THING.
>> GO AHEAD. AMY AFTER YOUR
COMMENTS WE WILL HAVE KARL AND
SUES AND HAVE TO BREAK FOR LUNCH
BREAK AND WILL COME BACK TO
ISSUES LATER IN THE DAY. AMY,
PLEASE.
>> IF I MAY ADD AND LATE TO
GETTING IT TO BOB. I CURATED A
FOUR-PAGE LIST OF RESOURCES OF
PUBLISHED ARTICLES ON PE
IMPLEMENTATION STRATEGIES.
THERE IS NOT MUCH OUT THERE.
WHAT THERE IS, IT HAS BEEN
CURATED BY ME AND WILL BE AN AMY
COMMENT SOMEWHERE IN THERE ABOUT
THAT.
HOPEFULLY THAT WILL BE
DISTRIBUTED TO YOU EITHER
ALREADY OR SOON THEREAFTER,
AFTER THIS PROGRAM.
>> WE HAVE HAD IT. IT IS IN TH
>> IT SOUNDS REALLY IMPORTANT
THAT WE BEGIN TO UNDERSTAND ONE
ANOTHER AND SEE ONE ANOTHER AND
TO GO OUT AND FIND BEST
PRACTICES MORE IMPORTANTLY
THINGS THAT DON'T WORK THAT IS
NOT SOMETHING PUBLISHABLE OR IF
IT IS, THEY HAVE WRITTEN THEIR
NOTES TO THEIR BOSS OR JUSTIFIED
A BUDGET OR WHATEVER. I CAN
TELL YOU PATIENT ENGAGEMENT IS
THE PROCESS. IT IS NOT AN
EXPENSE ITEM ITSELF. IT IS A
BETTER WAY OF DOING IT.
YOU HAVE TO HAVE A PROCESS BY
WHICH YOU CAN TAP INTO.
I WISH THERE WAS A POOL OF NIAMS
PATIENTS THAT ARE DEVELOPING --
YOU KNOW, DEVELOPING THE
CATEGORY. ARE YOU SKIN OR VAS
CLIETS OR HUMETOID ARTHRITIS OR
AFT YO POROSIS WHERE YOU CAN
PARTICIPATE. IT IS LIKE
CLINICAL TRIALS AND EVEN MORE
PARTICULAR TO INTERESTED AREAS
YOU MIGHT BE INTERESTED IN AND
AT SAME TIME THIS COULD BE A
GREAT REPOSTORY FOR
INVESTIGATORS TO TAP INTO. WE
ARE LOOKING FOR SOMETHING IN
ALASKA PATIENT PARTS NER /* --
PARTNERS -- THESE THAT NIAMS
COULD DEVELOP WOULD BE REALLY
HELPFUL. THANK YOU.
>> THANKS SO MUCH, AMY. WE WILL
HEAR FROM KARL AND SUES AND
BREAK FOR LUNCH.
I WILL CERTAINLY SO MUCH MORE TO
SAY AND LEARN HERE.
>> THANK YOU AND TO BOB AND
PANEL MEMBERS FOR INSPIRING
TALKS.
IT
IT'S A GOOD REMINDER, THE PRIMES
PEOPLE WHO ARE BENEFITING FROM .
QUICK QUESTION ABOUT THIS IDEA .
MAYBE THE PANEL TO SPEAK TO RESO
SUPPORT IT, I WOULD LIKE TO MAKD
SOMETIME DOWN THE ROAD ARE NOT Y
MAY NOT HAVE THE RESOURCES, WHAH
NIH TO HELP GET THOSE INSTITUTIY
ARE COMPETITIVE FOR THIS TYPE O
>> WE CAN WRITE THEM INTO THE BU
HAVE TO KNOW WHERE I START
>> I WANTED TO JUST SAY AGAIN TT
PCORI HAS THIS LAID OUT PRETTY
ARTFULLY AND SO IT IS BAKED INTO
THE PSA ITSELF SO THE
REQUIREMENTS ARE EXPLICITLY
LISTED, THERE'S BRAIN BEFORE
THEY APPLY, FOR PATIENT PARTNERS
BEFORE THEY APPLY.
THERE'S TRAINING AVAILABLE AFTER
YOU'RE AWARDED.
THE BUDGET IS EXPRESSLY -- IT
CALLS FOR ENGAGEMENT COSTS.
THE NEW EXPECTATION OF
ENGAGEMENT GUIDANCE I
REFERENCED, EVEN TALKS ABOUT
CONSIDERATIONS LIKE A POINT OF
CONTACT THAT'S ENGAGEMENT LEAD,
WHOSE JOB IT IS TO FIND
PATIENTS, CURATE, FACILITATE,
NURTURE THAT ELEMENT OF THE
STUDY.
AND THIS ONE BIG PICTURE
CONCEPT, AGAIN PCORI, PETER
TALKED ABOUT, DR. MERKEL TALKED
ABOUT HAVING BEEN ONE OF THE
PILOT AWARDEES, THE MOST
IMPORTANT PIECE OF ADVICE I
COULD GIVE HAVING BEEN AT THE
EARLY DAYS OF PCORI, DON'T
DABBLE.
GO DEEP.
YOU CAN BE NARROW BUT DON'T JUST
DIP YOUR TOE IN AND LET'S FIND A
COUPLE PATIENTS AND SEE WHAT
HAPPENS.
I WOULD PILOT.
TAKE ONE FUNDING ANNOUNCEMENT,
GO DEEP AND BE VERY INTENTIONAL
AND BAKE THE ENGAGEMENT FROM TIP
TO TOES, AND THEN LEARN FROM
THAT AND EXPAND GRADUALLYACROSS
THE FUNDING PORTFOLIO.
I THINK DABBLING ONLY REINFORCES
WHAT DOESN'T WORK BECAUSE IT'S
NOT MEANINGFUL.
SO YOU NEED TO REALLY THINK
ABOUT WHERE COULD WE PILOT, DO
THIS IN A VERY THOROUGH TRULY
INTEGRATIVE WAY.
I SEE MY FRIEND TIFFANY
MENTIONED LET'S PUT TOGETHER A
WORK GROUP OR COMMITTEE.
I WANT TO VOLUNTEER FOR THAT AS
WELL.
THIS IS MY PASSION, SETTING
THESE KINDS OF SYSTEMS UP.
I'M THRILLED THAT THIS
DISCUSSION IS HAPPENING.
THANKS SO MUCH.
>> YES, AND THANK YOU SO MUCH.
ON THAT NOTE LET ME JUST REMIND
THOSE WHO WERE HERE AT THE
BEGINNING BUT MISSED IT, WE'RE
PLANTING ON CONTINUING A DEEP
DIVE ON THIS TOPIC AS WELL AS
COMMUNITY ENGAGEMENT FOR THE
NEXT SEVERAL COUNCIL MEETINGS.
WE RECOGNIZE THERE'S A LOT OF
COMPLEXITY HERE, AND, YOU KNOW,
KIND OF HOW TO DO THIS IS NOT
NECESSARILY ALL MAPPED OUT FOR
INVESTIGATORS, SO WE LOOK
FORWARD TO WORKING WITH THOSE OF
YOU WHO ARE INTERESTED INCLUDING
OUR MUCH MORE EXTENDED GROUP OF
ADVOCACY ORGANIZATIONS WHO I'M
SURE COULD BE GREAT PARTNERS IN
THIS.
AND STARTING TO DEVELOP A PLAN
AND COMPILING THE NECESSARY
TOOLS AND GUIDANCE SO THAT OUR
INVESTIGATORS FEEL, YOU KNOW,
ENABLED, EMPOWERED TO DO THIS.
I WAS REMINDED OF SOME OF THE
CHALLENGES OUR INVESTIGATORS
HAVE FACED WHEN NEW DATA SHARING
AND MANAGEMENT GUIDELINES COME
OUT, OKAY, I GET IT, BUT HOW DO
I DO IT, RIGHT?
WE DON'T WANT TO PUT UP
REQUIREMENTS THAT OVERWHELM
INVESTIGATORS OR THAT MAKE THEM
FEEL UNABLE TO ADDRESS.
SO, THANK YOU SO MUCH TO OUR
THREE PRESENTER TODAY, SUZE,
AMY, AND WHERE IS PETER?
THERE'S PETER RIGHT THERE.
THANK YOU SO MUCH FOR YOUR
PRESENTATIONS AND YOUR
CONTRIBUTIONS AND I'M SURE WE'LL
BE CONTINUING TO ENGAGE ALL
THREE OF YOU AND MANY OTHERS
MOVING FORWARD.
WE'RE NOT DONE WITH THIS TOPIC.
THANK YOU SO MUCH.
AMY, THANK YOU FOR PUTTING THOSE
RESOURCES IN THE e-COUNCIL
BOOK.
THAT'S TERRIFIC.
SO, WE'RE GOING TO BREAK FOR
LUNCH, RIGHT, DARREN?
>> TAKE CARE.
>> THANK YOU SO MUCH.
AND.
>> TAKE CARE, EVERYONE.
>> THANK YOU, AMY AND SUZE.
>> THANK YOU SO MUCH.
>> MY UNDERSTANDING IS THAT YOU
ALL ARE GOING TO HAVE A SITE
VISIT TO THE NIH CAFETERIA,
WHICH ANN IS VERY EXCITED ABOUT.
YOU SAID YOU LOVE THE NIH
CAFETERIA.
SO, OKAY, I HOPE IT'S STILL --
BUT SO ANN KNOWS WHERE TO GO,
DOWN ON THE FIRST FLOOR.
SO, FOLLOW ANN.
I GUESS THE QUESTION IS DO YOU
ALL FULLY ENJOY THE AMBIANCE AND
HAVE YOUR LUNCH THERE OR DO YOU
WANT TO BRING IT TO THE SIXTH
FLOOR, WHAT'S THE PLAN?
>> THAT'S A GOOD QUESTION.
BEFORE WE FINALIZE THAT WE'LL
SEE WE'LL COME BACK AT 1:10.
>> 1:00.
>> 1:00?
WE'LL COME BACK AT 1:00 P.M. AND
WE'LL SEE EVERYBODY THEN, AND
THEN WE WILL PAUSE THE MEETING
FOR NOW AND FIGURE OUT THESE
LOGISTICS.
>> SO WE'RE GETTING READY TO GET
STARTED.
DR. CARTER IS GOING TO KICK OFF
THIS NEXT SESSION BECAUSE OF HIS
VERY SIGNIFICANT INVOLVEMENT IN
THE CLINICAL TRIALS WORKING
GROUP, BUT I WANTED TO POINT OUT
TO ALL OF YOU IN THE ROOM THAT
MAGICALLY AT THE BACK OF THE
ROOM A COFFEE CART ARRIVED.
SO, THANKS TO STEPHANIE HERNDON.
WE'RE GOING TO DO IT EVEN BETTER
NEXT TIME, AND SORRY TO THOSE OF
YOU WHO ARE JOINING VIRTUALLY,
BUT THOSE IN THE ROOM, HELP
YOURSELF.
>> CAN YOU HEAR ME OKAY?
>> YES.
SO, I WANT TO THANK JOAN BATHON,
WHO IS THE CHIEF OF RHEUMATOLOGY
AT COLUMBIA, ALONG WITH STUART
GOODMAN, WHO IS THE -- WAS AT
STANFORD, CO-CHAIRS OF THIS
WORKING GROUP.
JOAN MENTIONED THIS, THIS
WORKING GROUP HAS HAD 18
MEETINGS, AND THE CHAIRS HAD
ADDITIONAL 18 ON TOP OF THAT.
AND I WANT TO THANK CHUCK
WASHBAUM, DEONDRE AND DARREN, IN
SUPPORT OF THE WORKING GROUP.
SO, WITHOUT FURTHER ADO, THIS
GROUP DID -- STUART DID THE
INTERIM PRESENTATION IN AUGUST,
AND I THINK JOAN IS GOING TO
ILLUSTRATE THOSE BRIEFLY.
BUT WE'VE GOT A PRETTY BIG SLATE
OF THE COMMITTEE WORKING GROUP,
A BIG SLATE OF NEW
RECOMMENDATIONS.
GROUND RULES ARE THAT A WORKING
GROUP, THEY ARE FORMED AS A
WORKING GROUP OF COUNCIL, UNDER
THE STATUTORY AUTHORITIES FOR
GETTING INPUT ADVICE TO NIAMS.
PART OF THAT STATUTORY RULE IS
THE FINDINGS AND
RECOMMENDATIONS, QUOTE/UNQUOTE,
OF THE WORKING GROUP ARE
PRESENTED TO COUNCIL AND THAT
IT'S COUNCIL THAT ADVISES THE
INSTITUTE AS TO WHETHER THEY
WOULD ACCEPT AND PUT FORWARD
THOSE FINDINGS AND
RECOMMENDATIONS.
SO, DARREN, I DON'T KNOW IF
THERE'S A VOTE FOR THE COUNCIL
TO ACCEPT -- NO, NO VOTE, JUST
DISCUSSION AT THE END OF THE
PRESENTATION.
SO, WITH THAT, THANK YOU AGAIN
TO JOAN BECAUSE SHE REALLY HAS
DONE THE LION'S SHARE OF THE
WORK OVER THE LAST TWO MONTHS,
PULLING TOGETHER A VERY LARGE
SET OF RECOMMENDATIONS, A LOT
FROM PRETTY CRUDE TRANSCRIPTS OF
MEETINGS, FORMULATING INTO A
SERIES OF BULLET POINTS AND THEN
HONING THAT DOWN INTO WHAT
ACTUALLY SHE'S GOING TO PRESENT
TODAY WHICH ACTUALLY IS MORE
HIGHLIGHT AND ABBREVIATED FORM
OF A FULL SLIDE DECK WHICH IS IN
THE COUNCIL MEMBERS' ELECTRONIC
COUNCIL BOOK.
SO JOAN, THANK YOU.
>> THANKS, BOB.
I APPRECIATE THE OPPORTUNITY TO
PRESENT ON BEHALF OF STUART, WHO
IS SOMEWHERE IN ASIA, AND THE
COMMITTEE THAT I HAVE A LIST OF
AT THE END OF THE PRESENTATION
WHO DID AN ENORMOUS AMOUNT OF
WORK, AS BOB MENTIONED.
THE FORMAL CHARGE WAS HOW NIAMS
COULD SOLICIT AND FUND TRIALS
WITH THE GREATEST IMPACT IN
IMPROVING CLINICAL OUTCOMES
ACROSS OUR NIGH AMOUNTS
DISEASE -- SET OF DISEASES,
WHETHER RARE OR COMMON, AND
FOCUS ON IMPROVING PREVENTION,
DIAGNOSIS, AND TREATMENT OF
PEOPLE WITH THESE DISEASES AND
CONDITIONS.
AND THEN OF COURSE AS WE'RE
DOING TODAY TO PRESENT THESE
RECOMMENDATIONS TO THE ADVISORY
COUNCIL.
SO, SIX OF THE MAIN QUESTIONS
ARE CITED HERE, AND WE'LL GO
THROUGH ONE BY ONE, FIRST TWO
DEAL WITH TRIAL IMPACT, SECOND
DEAL WITH INVESTIGATOR AND
COMMUNITY ENGAGEMENT, THE LAST
TWO DEAL WITH TRIALS, BUDGETS,
AND SIZE.
THERE WERE ADDITIONAL
CONSIDERATIONS THAT WE AS A
WORKING GROUP CONSIDERED, AND
THOSE ARE QUESTIONS, TWO THREE,
AND FOUR OF THE QUESTION ONE WAS
ONE OF THE ORIGINAL SEVEN
QUESTIONS WHICH WE'LL GET TO.
I'LL REITERATE EACH AS WE GET
ALONG SO YOU DON'T HAVE TO
REMEMBER THEM.
AS BOB MENTIONED WE DID PRESENT
PRELIMINARY RECOMMENDATIONS TO
COUNCIL IN AUGUST, AND IT'S
EXCITE
EXCITING THAT THEY HAVE ALREADY
BEEN ADOPTED.
FIRST WAS THAT NIAMS SHOULD
CONSIDER DIFFERENT OPTIONS FOR
THE SMALL EXPLORATORY OR
FEASIBILITY CLINICAL TRIAL
MECHANISM THAT CURRENTLY
EXISTS -- THAT EXISTED AT THAT
TIME, R21, SHIFT TO ONE WITH
LONGER BUDGET AND TIME FRAME,
THE RATIONALE WAS EXISTING R21
WAS TOO SHORT AND BUDGET WAS TOO
SMALL TO EFFECTIVELY CONDUCT AN
EXPLORATORY CLINICAL TRIAL.
IN RESPONSE TO THIS
RECOMMENDATION NIAMS HAS ALREADY
DEVELOPED AND PUBLISHED A NEW
R61 SMALL EXPLORATORY TRIALS
NOTEBOOK TO REPLACE THAT R21,
DIRECT CAP FROM 400 TO 600,000
EXTENDED TO THREE-YEAR PERIOD,
NOW 12 PAGES ARE ALLOWED GIVING
MORE CHANCE TO DISCUSS THE
SCIENCE.
SO THAT'S VERY EXCITING.
THE SECOND RECOMMENDATION WAS
THAT NIAMS SHOULD CONSIDER
ESTABLISHING A TWO-FACE
MILESTONE-BASED AWARDS PROCESS
USING UG3/UH3 MECHANISM FOR
LARGE CLINICAL TRIALS.
AND THE BACKGROUND BRIEFLY WAS
THAT R 34 PLANNING GRANT
FOLLOWED BY U01 APPLICATION THAT
WAS THE PREVIOUS MECHANISM TO
GET A CLINICAL TRIAL APPROVED
AND FUNDED WAS A LONG PROCESS
WITH TWO SEPARATE SUBMISSIONS
AND TWO SEPARATE REVIEW GROUPS,
AND TWO REVIEW PROCESSES SO BY
THE TIME THE STUDY WAS FUNDED
THE IDEA FOR THE TRIAL MIGHT
HAVE GOTTEN A BIT STALE AND NOT
BE AS NOVEL AS IT ONCE WAS.
UG3/UH3 IS A COMPRESSED
MECHANISM WHICH REQUIRES ONE
APPLICATION, AND THEN IF THE
PERSON MEETS MILESTONES THEY
PROGRESS SEAMLESSLY INTO THE
UH3.
I'M HAPPY TO SAY NIAMS ACCEPTED
THIS RECOMMENDATION AND HAS
DEVELOPED AND PUBLISHED JUST IN
THE LAST COUPLE DAYS A NEW
UG3/UH3 PHASED CLINICAL TRIALS
NOFO, VERY EXCITING, WE'RE HAPPY
THAT RECOMMENDATION WAS FOUND TO
BE GOOD.
SO LET US FOCUS ON THE NEW
RECOMMENDATIONS TODAY.
AND AGAIN JUST REMINDING OF THE
THREE AREAS WE'LL TALK ABOUT
NOW, TRIAL IMPACT, COMMUNITY
ENGAGEMENT, AND BUDGETS AND
SIZES OF TRIALS.
FIRST QUESTION, WHAT TYPES OF
CLINICAL TRIALS HAVE THE
GREATEST IMPACT ON PEOPLE'S
LIVES.
AND WE STRONGLY FELT AS A
WORKING GROUP NIAMS SHOULD
CONSIDER SHIFTING STRATEGY FOR
MAXIMIZING NUMBER OF FUNDED
TRIALS TO FOCUS ON FUNDING THE
MOST IMPACTFUL TRIALS.
AND WHAT IS IMPACTFUL?
OBVIOUS CRITERION MIGHT BE A
TRIAL THAT PRODUCES FINDINGS
LIKELY TO INFORM TREATMENT
GUIDELINE.
THE SECOND RECOMMENDATION IS
NIAMS SHOULD PRIORITIZE SIMPLE
TRIALS THAT EMPHASIZE IMPACT OF
INTERVENTION, TAKING INTO
ACCOUNT THE EFFECT SIZE OF THAT
INTERVENTION AND BURDEN OF
DISEASE, BOTH AT INDIVIDUAL AND
POPULATION LEVELS.
TO TRANSLATE THAT, FOR EXAMPLE,
INTERVENTIONS THAT HAVE THE
POTENTIAL TO HAVE A VERY
PROFOUND IMPACT IN A RARE
DISEASE SHOULD IDEALLY BE
BALANCED WITH INTERVENTIONS THAT
MIGHT HAVE SMALLER EFFECTS BUT
APPLIED TO A VERY COMMON
DISEASE.
AND THE THIRD RECOMMENDATION WAS
THAT NIAMS SHOULD ALIGN WITH
RELEVANT PROFESSIONAL AND
PATIENT COMMUNITIES TO BEST
UNDERSTAND THE VIEWS OF THESE
STAKEHOLDERS OF THEIR BIGGEST
UNMET NEEDS.
WE'RE GOING TO COME BACK TO THIS
THEME LATER, POINT NUMBER 3.
HOW DOES ONE JUDGE THE IMPACT OF
A CLINICAL TRIAL?
WE DO THAT TO SOME EXTENT DURING
STUDY SECTION REVIEWS, BUT
WORKING GROUP FELT VERY STRONGLY
THAT TO BETTER ASSESS TRIAL
IMPACT NIAMS SHOULD CONSIDER
FORMAL ROLE FOR COUNCIL, THIS
ADVISORY COUNCIL AS A SECOND
LEVEL OF REVIEW.
THE RATIONALE FOR THAT, STUDY
SECTIONS ARE EXCELLENT AT
SCORING BUT IMPACT POSSIBLY
SLIPS THROUGH THE CRACKS IN THE
FINAL RECOMMENDATIONS.
WE THOUGHT NIAMS COUNCIL WAS
OPTIMALLY POSITIONED TO CONDUCT
SECOND LEVEL, GREAT EXPERTISE IN
THE ROOM AND ONLINE REPRESENTING
MULTIPLE DISEASE AREAS, AND
DIFFERENT GROUPS, BOTH
PHYSICIANS, PhDs, AND
PATIENTS, ET CETERA.
AND ALSO THIS COULD EASILY BE
CONDUCTED AND VOTED ON DURING
YOUR COUNCIL MEETINGS.
SO IT WOULD BE UP TO OBVIOUSLY
NIAMS TO DEVELOP A PROCESS IF
THEY ADOPTED THIS RECOMMENDATION
BUT WE THOUGHT ONE WAY TO DO
THIS WOULD BE THAT THE COUNCIL
COULD IDENTIFY OR -- SORRY,
PROGRAM OFFICERS COULD IDENTIFY
ALL TRIAL APPLICATIONS WITH
BUDGET GREATER THAN $500,000 OR
GREATER PER YEAR AND TARGET FOR
REVIEW AT THE MEETING.
THE P.O.s WOULD WRITE UP A
SHORT SUMMARY OF THE TRIAL AND
ITS POTENTIAL IMPACT, TWO OR
THREE COUNCIL MEMBERS WOULD BE
ASSIGNED TO REVIEW THAT
APPLICATION BEFORE THE COUNCIL
MEETING.
IT HAS ACCESS TO P.O.'S
WRITE-UP, APPLICATION AND STUDY
SECTION'S REVIEW.
AND THEY WOULD BE ASKED TO WRITE
A SMALL SUMMARY AND
RECOMMENDATION AND PRESENT IT AT
THE COUNCIL MEETING AND THE
COUNCIL COULD VOTE ON THIS AT
THAT TIME.
SO IT DOES ADD EXTRA WORK TO
COUNCIL BUT WE THOUGHT THAT
COULD BE A VERY HELPFUL WAY TO
REALLY WEED OUT OR SELECT OUT
TRIALS WITH THE GREATEST IMPACT.
SECOND YES, WHAT IS NIAMS REACH
IN THE LANDSCAPE, AGAIN THINKING
ABOUT IMPACT, THE GROUP FELT
NIAMS SHOULD EMPHASIZE STUDIES
THAT ADVANCE OR IMPACT CLINICAL
MANAGEMENT AND GUIDELINES.
THE NIAMS UNIQUE NICHE IS
DEFINED BY AREAS WE THOUGHT ARE
NOT FUNDED BY INDUSTRY WITH
LIMITED BUDGET OF NIAMS, IT'S
IMPOSSIBLE TO COMPETE FOR
INDUSTRY-SPONSORED LARGE DRUG
TRIALS, FOR EXAMPLE.
LASTLY, WE THOUGHT NIAMS SHOULD
CONSIDER TRIAL SELECTION PROCESS
WITH SPECIFIC CRITERIA FOR
GAME-CHANGING RESEARCH IN THE
VARIOUS NOFOs, GAME-CHANGING
MEANING HIGH IMPACT, THINGS THAT
WILL CHANGE THE LIVES THE
PATIENTS WITH OUR ILLNESSES.
THE THIRD QUESTION REFERRED TO
REACHING OUT TO THE EXTRAMURAL
COMMUNITY, SHOULD NIAMS SOLICIT
TOPICS TO PRIORITIZE FROM THE
EXTRAMURAL COMMUNITY.
WE AGREED, YES, INPUT SHOULD BE
SOUGHT IN THE FULL RANGE OF
AREAS OF THE NIAMS MISSION FROM
A BROAD ARRAY OF STAKEHOLDERS,
THESE WOULD INCLUDE ACADEMIA,
PROFESSIONAL SOCIETIES,
VOLUNTARY ORGANIZATIONS, AND
COMMUNITY GROUPS SUCH AS THOSE
REPRESENTED IN THE NIAMS
COALITION.
AND WE REALIZE YOU DO A LOT OF
THIS ALREADY BUT IT COULD BE
MORE FORMALIZED AND EXPANDED.
WE THOUGHT STRONGLY INTERACTIONS
WITH COMMUNITY GROUPS SHOULD
BIDIRECTIONAL AND OBTAINED IN
COORDINATED AND SUSTAINED
FASHION.
THE GOAL OF THIS IS TO FOSTER
AND BUILD COMMUNITY'S CONFIDENCE
IN CLINICAL RESEARCH PROCESS,
WOULD RESULT AT LEAST IN
BUILDING CONFIDENCE IN THE
RESEARCH PROCESSES.
NIAMS SHOULD IDENTIFY AND
PUBLISH KEY AREAS OF INTEREST
AND SUPPORT INVESTIGATORS
PURSUING NOVEL APPROACHES THAT
ADDRESS THE RESEARCH GAPS THAT
WERE DEFINED IN CONCERT WITH
EXTRAMURAL COMMUNITY.
LASTLY, RECOMMEND AS ALREADY
DONE, DISSEMINATE NIAMS AREA OF
INTEREST BY LISTING THEM ON THE
WEBSITE AND ISSUING NOSIs AND
NOFOs.
FOURTH QUESTION HOW CAN NIAMS
BETTER SUPPORT CLINICAL TRIAL
APPLICANTS, A VERY LARGE LIST OF
BULLET POINTS HERE BUT WE TRIED
TO CONDENSE THEM AND DIVIDED
THEM INTO TWO SECTIONS, ONE IS
BY DEVELOPING AND PROMOTING
INFORMATIONAL AND EDUCATIONAL
INITIATIVES, THE OTHER IS BY
EXPANDING NIAMS INSIGHT.
THE FIRST ONE, WE KNOW NIAMS
DOES A NICE JOB OF PROVIDING
ACCESS TO A LOT OF INFORMATION,
SOMETIMES IT'S HARD TO FIND OR
HASN'T BEEN UPDATED IN SOME
CASES.
SO WE ARE PUTTING FOUR MAJOR
BULLET POINTS HERE THAT I THINK
WE THOUGHT WOULD HELP.
ONE OF THE MOST IMPORTANT IS
ENCOURAGING APPLICANTS TO
COMMUNICATE EARLY WITH PROGRAM
DIRECTORS.
WHILE THE PROGRAM DIRECTORS DO
THOSE, THEY ARE REALLY COMMITTED
TO THIS, BUT WE ALL HAVE
EXPERIENCES WITH JUNIOR
INVESTIGATORS THINKING THAT NIH
FOLKS ARE OFF LIMITS, THEY ARE
NOT ALLOWED TO TALK TO THEM.
SO WE DO AT OUR END ENCOURAGE
THEM TO ALWAYS CONTACT THE
P.O.s AT NIAMS BUT I THINK IF
IT'S A LITTLE BIT MORE OBVIOUS
AND IT'S PROMOTED BROADLY, IN
WHATEVER COMMUNICATIONS YOU WANT
TO USE, THAT WOULD BE REALLY
HELPFUL.
SECOND TO CONDUCT WEBINARS AND
COMMUNICATION FOR NEW TRIAL
NOFOs, TO A LARGE EXTENT BUT
PUTTING THAT IN PRINT.
PROVIDE TOOLS FOR INVESTIGATORS,
CENTRAL AREAS WHERE INFORMATION
FOR CLINICAL TRIALS IS EASILY
FOUND.
THIS COULD INCLUDE LESSONS
LEARNED, TOOLS FOR DATA
REPOSITORIES, COMPILE
INFORMATION THAT INVESTIGATORS
COULD BE USED TO IDENTIFY SITES
AND DEVELOP GUIDANCE FOR
INVESTIGATORS ON FUNDING
PARTNERSHIPS.
WE HAVE TO GET PERMISSION FOR
CLINICAL TRIAL APPLICATION THAT
WILL REQUEST GREATER THAN
$500,000 PER YEAR, AND
OFTENTIMES PARTNERSHIPS WITH
OTHER FUNDING ORGANIZATIONS OR
OTHER INSTITUTES ARE NECESSARY
IN ORDER TO MOVE A TRIAL
FORWARD, A VERY OPAQUE PROCESS,
INVESTIGATORS DON'T KNOW HOW TO
APPROACH, ESPECIALLY JUNIOR
INVESTIGATORS.
AND WE KNOW THE NIAMS FOLKS ARE
ANXIOUS TO HELP INVESTIGATORS
WITH THIS SO MAYBE DEVELOPING,
ACKNOWLEDGING THIS SOMEWHERE IN
A GUIDANCE DOCUMENT, AND
DEVELOPING SOME TIPS FOR THE
INVESTIGATORS TO ACCESS WOULD BE
GREAT.
NIAMS COULD BETTER SUPPORT
CLINICAL TRIAL APPLICANTS BY
EXPANDING NIAMS OVERSIGHT.
THE FIRST RECOMMENDATION IS
REALLY ABOUT THE REVIEW PROCESS.
AND WE SUGGEST THAT NIAMS EXPAND
THE SCOPE OF THE AMSC STUDY
SECTION TO INCLUDE SOME OTHER
CLINICAL MECHANISMS, CLINICAL
STUDY MECHANISMS.
EXAMPLE WOULD BE FOR EXAMPLE
ORTHOPEDICS STUDIES, LARGE
OBSERVATIONAL STUDIES WHERE
OUTCOMES ARE BEING ASSESSED,
WHETHER THEY ARE GOOD OUTCOMES
OR BAD OUTCOMES, IDEAL
OBSERVATIONAL STUDIES TO
IDENTIFY RISK FACTORS FOR GOOD
VERSUS BAD OUTCOMES BUT THESE GO
THROUGH THE CSR AND GET ASSIGNED
TO A STUDY SECTION THAT THE CSR
OVERSEES, AND THE EXPERTISE FOR
THESE TYPES OF STUDIES IS JUST
NOT THERE IN THE STUDY SECTIONS
SO WE THOUGHT IT WOULD BE
REASONABLE TO LOOK AT SOME OF
THESE AND CREATE A MECHANISM TO
BRING OBSERVATION STUDIES OR
MECHANISMS INTO THE PURVIEW OF
THE AMSC.
A SECOND RECOMMENDATION TO
DEFINE AND FURTHER EXPAND
PROGRAM STAFF ROLES IN CLINICAL
TRIAL OVERSIGHT, THIS WILL OCCUR
IN THE UG3/UH3 PROCESS BECAUSE
THERE'S CLOSER INVOLVEMENT FROM
PROGRAM STAFF.
BUT TWO VERY MAJOR
RECOMMENDATIONS UNDER THIS POINT
ARE, ONE, ESTABLISH SUBSPECIAL
SHY TEAMS OF PROGRAM DIRECTORS
TO OVERSEE TRIALS BASED ON TRIAL
TYPE, FOR EXAMPLE COULD BE A
GROUP OF PROGRAM STAFF THAT
SPECIALIZE IN DRUG INTERVENTION
TRIALS.
ANOTHER ONE ON SURGICAL
INTERVENTION TRIALS.
A THIRD ON BEHAVIORAL
INTERVENTION TRIALS.
AND THOSE SPECIALTY GROUPS
COULD -- THAT EXPERTISE WOULD GO
ACROSS ALL DISEASE AREAS.
A SECOND REALLY IMPORTANT ONE IS
TO ADD MORE M.D.s TO THE
PROGRAM DIRECTOR STAFFING POOL.
I KNOW IT'S BEEN A CHALLENGE AND
WE FULLY RESPECT ALL THE
PhDs IN THE PROGRAM STAFF,
HOW HELPFUL THEY ARE IN OUR
CLINICAL TRIALS PROCESSES.
BUT THEY WERE IDENTIFIED TO BE
GAPS IN THE CLINICAL KNOWLEDGE
AND BACKGROUND AND EVEN COMFORT
WITH THAT AMONGST SOME OF THE
PhD staff.
SO ADDING SOME M.D.s WOULD BE
BENEFICIAL.
A THIRD RECOMMENDATION TO
EVALUATE AND IDENTIFY THE MOST
IMPACTFUL TRIAL DESIGNS FROM THE
INFORMATIONAL WEBSITE.
FOURTHLY PROVIDE EDUCATIONAL
RESOURCES FOR FDA REGULATED
STUDIES, THAT ARE THE RULES AND
REGS FOR EXEMPT PROTOCOLS FROM
FDA REGULATION, FOR EXAMPLE.
A LOT HAS BEEN DEVELOPED FOR THE
HEAL INITIATIVE, THAT COULD BE
TRANSPORTED INTO THE NIAMS
PORTFOLIO FOR INVESTIGATORS.
ALL RIGHT.
MOVING ON, THE NEXT TWO
QUESTIONS HAVE TO DO WITH THE
TRIAL BUDGETS, WHAT IS
APPROPRIATE BUDGET CEILING AND
SHOULD NIAMS SUPPORT FEWER
TRIALS WITH LARGER BUDGETS OR
CONTINUE TO SUPPORT A BROADER
MIX OF TRIALS.
SO WE ALL UNANIMOUSLY AGREED
NIAMS SHOULD CONTINUE TO SUPPORT
A BROAD MIX OF CLINICAL TRIAL
MECHANISMS INCLUDING RANGE OF
BUDGETS AND RANGE OF TRIAL
DURATIONS.
BUT WE DID FEEL, BULLET POINT
NUMBER 2, WE SHOULD CONSIDER
REISSUING A FUNDING OPPORTUNITY
FOR CLINICAL TRIAL PLANNING
GRANT THAT FOCUSES ON AN EARLIER
STAGE THAN UG3.
NOW THAT WE HAVE THE UG3 AND
UH3, UG3 IS A PLANNING MECHANISM
THAT'S VERY PROXIMATE TO
INITIATION OF TRIAL, ALMOST
SHOVEL READY, NEEDING TO DO A
FEW CRITICAL THINGS DURING THAT
PLANNING PHASE AND THEN
SEAMLESSLY WILL GO IMMEDIATELY
INTO THE CLINICAL TRIAL.
BUT WE NEED A MECHANISM WHERE
THE CLINICAL TRIAL ISN'T QUITE
SO MATURE, THERE NEEDS TO BE AN
EXPERT GROUP ASSEMBLED TO WORK
OUT PROTOCOL DEVELOPMENT,
INCLUSION/EXTRUSION CRITERIA,
IDENTIFY SIDES THAT COULD
PARTICIPATE, THINGS LIKE THAT.
WE THINK THAT WOULD BE HELPFUL
AS EARLIER PHASE PLANNING GRANT
AND THEN IT SHOULD, AGAIN,
INCLUDE IMPACT AS A REVIEW
CRITERION.
SO THIS POTENTIAL CLINICAL TRIAL
IS GOING TO BE DISCUSSED, IS IT
WORTH DOING, IS IT GOING TO BE
IMPACTFUL.
THE COMMITTEE FELT THAT
RECOMMENDING A BUDGET CEILING,
BUDGET THRESHOLD IS NOT IN OUR
PURVIEW BECAUSE THERE ARE
COMPLICATED TRADEOFFS, HAVE YOU
INCREASE THIS YOU HAVE TO
DECREASE SOMETHING ELSE AND
DON'T HAVE AN IN-DEPTH
KNOWLEDGE, SO WE DID DEFER ON
THAT QUESTION.
THE ADDITIONAL QUESTIONS THAT WE
AS A CLINICAL TRIALS WORKING
GROUP ADDED ARE 2, 3, 4, OUR
LAST ONES.
SO SHOULD NIAMS EXPLORE
ESTABLISHING OR FACILITATING
CLINICAL TRIALS NETWORK?
HOW CAN NIAMS SUPPORT DIVERSITY
IN ENROLLMENT AND HOW CAN NIAMS
ENGAGE THOSE WITH LIVED
EXPERIENCES IN THE CLINICAL
TRIALS PROCESSES?
SO, WE SPEND A LOT OF TIME
THINKING WHETHER NIAMS SHOULD
FACILITATE OR ESTABLISH A
NETWORK.
THE RATIONALE IS RIGHT NOW EVERY
TIME A CLINICAL TRIAL IS FUNDED
BY NIAMS THAT INVESTIGATOR HAS
TO REINVENT THE WHEEL FOR EVERY
PROCESS, AND IT'S INEFFICIENT
AND COSTLY.
AND FOR FIRST-TIME CLINICAL
TRIAL APPLICANTS, DE NOVO
DEVELOPMENT OF ALL THESE
NECESSARY TRIAL ELEMENTS AND
INFRASTRUCTURE ELEMENTS CAN BE
VERY, VERY OVERWHELMING.
AND IT MAY DIMINISH THEIR
ENTHUSIASM TO PURSUE WHAT MIGHT
BE A VERY IMPACTFUL TRIAL
APPLICATION.
SO, WE DID RECOGNIZE HOWEVER
THAT BECAUSE OF THE BROAD
MISSION AREA OF NIAMS THAT
COVERS SKIN, ORTHOPEDICS,
MUSCLE, AND AUTOIMMUNE DISEASE,
IT IS SO BROAD AND THE BUDGET IS
SO LIMITED THAT IT WOULD BE
VIRTUALLY IMPOSSIBLE TO DEVELOP
A MATURE COMPLETE CLINICAL
TRIALS NETWORK.
SO OUR RECOMMENDATION INSTEAD IS
FOR NIAMS TO CONSIDER WHETHER
THEY ARE AT LEAST A COMPONENT OR
TWO OR THREE OF AN EXISTING CTN
OR CTSI THAT COULD PROVIDE
ECONOMIES OF SCALE AND EFFORT SO
TRIALISTS COULD PLUG INTO THAT
AND NOT HAVE TO GENERATE DE
NOVO.
SOME COMPONENTS INCLUDE BUT NOT
LIMITED TO CENTRALIZED DATA
COORDINATING CENTERS, DATA
COLLECTIONS, SUPPORT FOR
RECRUITMENT, DSMBs WHICH ARE
ALREADY PROVIDED AND I THINK
EVERYBODY APPRECIATES, SINGLE
IRBs, NAVIGATOR NETWORKS, DATA
SHARING RESOURCES, REGULATORY
SUPPORT AND CLINICAL TRIAL
DESIGN, A LOT TO THINK ABOUT.
NEXT QUESTION HOW CAN NIAMS
SUPPORT DIVERSITY IN CLINICAL
TRIAL ENROLLMENT?
SO ONE SUGGESTION WAS THAT NIAMS
SHOULD DEVELOP A LIST OF SITES
THAT HAVE ESTABLISHED EXPERTISE
AND COMMUNITY ENGAGEMENT AND
MAKE LISTS AVAILABLE TO
APPLICANTS OR GRANTEES TO
PARTNER WITH THOSE SITES.
FOR EXAMPLE, INVESTIGATOR WHO IS
DOING A CLINICAL TRIAL PROPOSING
A CLINICAL TRIAL IN HIS OR HER
AREA OF THE COUNTRY WHICH
HAPPENS TO BE 95% CAUCASIAN
COULD ACCESS THIS LIST, IDENTIFY
SOME SITES THAT HAVE A TRACK
RECORD IN ENROLLMENT OF DIVERSE
POPULATIONS, ASK THEM TO
PARTICIPATE WITH SUB-AWARDEES,
THAT WOULD FACILITATE A MORE
DIVERSE ENROLLMENT.
SECOND SUGGESTION, NIAMS SHOULD
OFFER MORE TRAINING TO SROs
AND REVIEWERS HOW TO EVALUATE
APPLICATIONS FOR DIVERSITY OF
ENROLLMENT.
FOR EXAMPLE, IF AN INVESTIGATOR
PLANS ON ENROLLING CLINICAL
TRIAL POPULATION THAT REFLECTS
THEIR CLINIC OR GEOGRAPHIC
POPULATION, FOR EXAMPLE, 95%
CAUCASIAN AGAIN, THEN THERE WILL
NOT BE DIVERSITY AND THEY SHOULD
BE -- REVIEWERS AND SROs
SHOULD HAVE A WAY TO COMMENT ON
THAT AND SUGGEST SOME
MODIFICATIONS.
WAYS NIAMS COULD FACILITATE
DIVERSITY ENROLLMENT BY SHARING
OPTIONS, LESSONS LEARNED, BEST
PRACTICES AGAIN, SHARING
APPROACHES FROM DIFFERENT
INSTITUTIONS, APPROACHES TO
RECRUITMENT OF DIFFERENT
CULTURES, GUIDE APPLICANTS TO
RESOURCE AND PRACTICES.
AND THEN IMPORTANTLY OFFER
GUIDANCE ON BUDGETING.
WE FELT NIAMS SHOULD VERY
OVERTLY ENCOURAGE THE ENGAGEMENT
OF INDIVIDUALS WITH LIVED
EXPERIENCES WITH A DISEASE FROM
THE VERY START OF CLINICAL
TRIALS WITHIN THE DESIGN PHASE
TO IMPLEMENTATION PHASE AND THIS
DISSEMINATION PHASE.
THIS ENGAGEMENT COULD EXTEND TO
CURRENT PARTICIPANTS IN ACTIVE
CLINICAL TRIALS, AND THEIR
INVOLVEMENT COULD BE AT ANY
NUMBER OF LEVELS, FOR EXAMPLE
ADVISING ON RECRUITMENT,
RETENTION AND AWARENESS, SUMMARY
EVENTS REPORTED, RETURNING
INDIVIDUAL RESULTS IF
APPROPRIATE, ENGAGE ON UPDATES
OF TRIAL PROGRESS.
SO THAT IS THE END OF THE
RECOMMENDATIONS.
I JUST WANTED TO REVIEW -- BOB
SUMMARIZED, OUR TIMELINE WAS 12
MONTHS, EXTENDED TO 18 MONTHS
BECAUSE OF ALL THE QUESTIONS AND
ISSUES THAT WE NEEDED TO
DISCUSS.
WE DID A VERY DEEP DIVE INTO THE
NIAMS PORTFOLIO WITH SHANARD,
BOB, CHUCK, LOOKED AT CLINICAL
TRIAL MECHANISMS THAT NIAMS HAS,
THE NUMBER OF APPLICATIONS PER
YEAR IT RECEIVED, PERCENT
FUNDED, TRENDS OVER YEARS, ALL
OF THESE ASPECTS, THE NUMBER OF
TRIALS THAT PUBLISH RESULTS AND
HOW IMPACTFUL THEY WERE.
WE HAD PRESENTATIONS BY AND
DISCUSSIONS WITH MULTIPLE NIH
INSTITUTES ABOUT THEIR OWN
CLINICAL TRIAL MECHANISMS, WHAT
WORKED, WHAT DIDN'T WORK.
WE TALKED ABOUT CLINICAL TRIALS
NETWORK, WHAT WORKED, WHAT
DIDN'T WORK.
THAT WAS VERY HELPFUL IN
INFORMING OUR OWN
RECOMMENDATIONS.
THEN AMONGST OURSELVES WE HAD
EXTENSIVE DISCUSSIONS AND
DEVELOPED OUR CURRENT
RECOMMENDATIONS, WE HAD A
PROCESS TO GET TO CONSENSUS AND
I CAN TELL YOU ALL OF THE
RECOMMENDATIONS TODAY WERE
AGREED UPON BY ALL OF THOSE, SO
MEMBERS OF THE COMMITTEE.
LASTLY I WOULD BE REMISS IF I
DIDN'T SHOUT OUT TO ALL OF OUR
NIAMS COLLEAGUES AND
FACILITATORS WHO ARE LISTED AS
WELL.
BOB AND CHUCK AND DEIDRE.
THIS IS A LIST OF EVERYONE WHO
PARTICIPATED.
STUART GOODMAN WAS CO-CHAIR OF
THIS PROCESS, AND COULDN'T BE
HERE TODAY.
REPRESENTING ORTHOPEDICS,
PATIENT ADVOCACY, RHEUMATOLOGY,
DETERMINE NOTHING, MUSCLE, BONE,
AND PAIN AND BEHAVIORAL, ALL THE
MISSION AREAS WERE COVERED IN
OUR GROUP.
LASTLY THANKS TO ALL OF THE
NIAMS FACILITATORS AND STAFF
SUPPORT, ESPECIALLY BOB AND
CHUCK, MORE RECENTLY DANDRE,
DARREN, AND TOM, OUR BOY FRIDAY,
WHO DID THE MINUTES,
ORGANIZATIONS OF THE MEETINGS
SCRIBE AND SLIDE GENERATOR.
THAT IS THE END.
I WILL STOP SHARING.
UNLESS YOU WANT ME TO LEAVE THE
SLIDES UP.
I'LL STOP SHARING.
>> SO, JOAN, LET ME FIRST START
OFF HERE AND PROFUSELY THANK YOU
AND STUART AND ALL OF YOUR
COLLEAGUES, ALL OF THE STAFF.
WHAT AN INCREDIBLE RESOURCE FOR
US.
THE TIME YOU TOOK.
I HAD ASKED SOMEONE TO CALCULATE
ALL THE OURS, COLLECTIVE HOURS,
I DON'T KNOW, THOUSANDS.
BUT WE'RE SO GRATEFUL.
EACH ONE OF THESE QUESTIONS IS
SO IMPORTANT.
AND YOU TOOK SUCH A THOUGHTFUL
COMPREHENSIVE APPROACH.
WE'RE GOING TO SPEND A LOT OF
TIME GOING THROUGH ALL OF THESE
AND FIGURING OUT HOW TO
INCORPORATE THEM, HOW TO TAKE
YOUR RECOMMENDATIONS, BUT I
WANTED TO THANK YOU AND WE HAVE
OTHERS HERE, MARIA WHO WE'LL
HEAR FROM IN A MINUTE, BUT
REALLY GRATEFUL.
I CAN'T SAY THAT ENOUGH.
SUCH AN IMPORTANT TOPIC FOR US.
ON THAT NOTE I'LL TURN IT TO BOB
TO GO FROM HERE.
>> I WANT TO GIVE MARIA AND JOSH
JACOBS, A FORMER COUNCIL MEMBER,
AS THEY SAY AROUND HERE ONCE A
COUNCIL MEMBER ALWAYS A COUNCIL
MEMBER.
MARIA, JOSH, WHAT WOULD YOU LIKE
TO ADD?
>> THANK YOU VERY MUCH.
IT WAS A PLEASURE WORKING WITH
JOAN AND STUART, AND GOING IN
SUCH DEEP EXPLORATION, WHAT WAS
AVAILABLE, WHAT COULD BE DONE
HAS BEEN AN ENLIGHTENING
PERSPECTIVE.
I KNOW YOU'RE ALL CONSTRAINED BY
POWERS ABOVE YOU, WHAT YOU CAN
DO, AS FAR AS CHANGING THE
PORTFOLIO, BUT I HOPE THAT SOME
OF THESE RECOMMENDATIONS CAN BE
MOVED FORWARD.
>> WELL, WHAT I WOULD ADD IS IT
WAS A VERY EFFECTIVE PROCESS.
I WAS VERY IMPRESSED WITH THE
GROUP ASSEMBLED, TASK FORCE,
CONCERNING BREADTH AND DEPTH OF
EXPERTISE IN THIS AREA.
I WOULD JUST STRESS AS JOAN SAID
THAT WE'RE -- WE STRESSED
IMPORTANCE OF CLINICAL TRIAL
PORTFOLIO OF NIAMS, IT HAS
POTENTIAL TO REALLY ADVANCE OUR
VARIOUS FIELDS.
FINALLY, JOAN WAS CORRECT;
DESPITE SOME DIFFERENT
PERSPECTIVES THAT WE ALL BROUGHT
TO THE TABLE, WE LEFT WITH OUR
LAST MEETING ALL WITH CONSENSUS
RECOMMENDATIONS SO I THINK THAT
THE PROCESS WAS VERY EFFECTIVE,
HATS OFF TO JOAN AND STUART, AND
THE NIH LEADERSHIP FOR
CONDUCTING A VERY EFFECTIVE
PROCESS.
>> KURT, DID YOU WANT TO
COMMENT?
>> HE'S THE SHY ONE.
>> NO, NO, I JUST JOINED RIGHT
NOW, I JUST POPPED IN.
I HEARD JOSH'S COMMENTS,
CERTAINLY ECHO IT.
IT WAS REALLY A LOT OF FUN AND
WORK, AN INFORMATIVE THING TO BE
ON THE WORKING GROUP AND TRY TO
CREATE RECOMMENDATIONS WORKING
WITH THE WHOLE TEAM AND BOB
CARTER.
>> TIFFANY WAS ON FOR THE
PATIENT ENGAGEMENT THIS MORNING,
WHICH IS INTERESTING.
I DON'T KNOW IF SHE PURPOSELY
JOINED THAT PART OF THE SESSION.
ALSO PETER WAS ALSO ON.
ALL RIGHT.
WE'LL OPEN UP THE FLOOR.
WE HAVE PLENTY OF TIME.
COMMENTS?
THINGS WE SHOULDN'T DO?
>> I'LL SAY SOMETHING ELSE.
I REALLY APPRECIATED THE FACT
THAT THIS WASN'T JUST PIE IN THE
SKY BUT YOU RECOGNIZED SOME OF
THE CONSTRAINTS WE HAVE AND THAT
OBVIOUSLY INFLUENCED YOUR
RECOMMENDATIONS.
AND THAT PUTS US SO MUCH FARTHER
TO BEING ABLE TO WORK WITH YOUR
RECOMMENDATIONS.
SO THANK YOU FOR INCLUDING THAT
REALLY IMPORTANT CONTEXT AS
WELL.
>> TWO QUESTIONS.
ONE IS, AND FOR COMMITTEE
MEMBERS, WHERE DO THESE
RECOMMENDATIONS STAND RELATIVE
TO WHAT OTHER INSTITUTES ARE
DOING?
IS THIS THE BEST OF THE BEST?
OR SOMEWHERE IN THE MIDDLE OR
BABY STEPS?
THAT'S MY FIRST QUESTION.
AND THE SECOND ONE IS RELATED TO
THAT IN TERMS OF WHAT'S THE NEXT
STEP IN TERMS OF FEASIBILITY AND
IMPLEMENTATION, PHASING, HOW DO
WE ENVISION THAT?
>> I CAN ANSWER THE FIRST ONE.
I'LL DEFER TO THE NIAMS FOLKS
FOR THE SECOND.
WHERE DO RECOMMENDATIONS STANDS
WITH OTHER INSTITUTES, YEAH, WE
HAVE SUCH A SMALL BUDGET
COMPARED TO THE OTHER INSTITUTES
THAT WE TALKED TO, FOR THE MOST
PART.
SO EVEN SO, WE HEARD ABOUT THEIR
UG3/UH3 SUCCESSES, HOW MUCH MORE
STREAMLINED IT WAS AND HOW MUCH
INVOLVEMENT THE NIAMS STAFF
WOULD HAVE, AND SO WE ARE RIGHT
IN LINE WITH THEM BY TAKING
THEIR -- WE ADOPTED THEIR
METHODS FOR THE BIG TRIALS.
AND SO I THINK WE'RE RIGHT IN
LINE WITH A LOT OF THE
INSTITUTES IN THAT REGARD.
AS FAR AS, YOU KNOW, BUDGETING
THOUGH THAT LIMITS WHAT WE CAN
DO IN THE CLINICAL TRIALS
NETWORK END OF THINGS I THINK,
AND THAT WOULD HAVE BEEN A NICE
AREA TO BE ABLE TO MAKE SOME
STRONGER RECOMMENDATIONS, THAT
WAS CONSTRAINED BY BUDGETS.
WHEREAS THE OTHER BIG INSTITUTES
WITH A LOT OF MONEY, CLINICAL
TRIALS DO HAVE VERY DEVELOPED IN
SOME CASES CLINICAL TRIALS
NETWORKS.
THAT'S FOR LINDSEY ET AL.
>> I WAS GOING TO BRIEFLY ADD
WITH RESPECT TO NETWORKS THOUGH
TWO THINGS THAT MIGHT BE
HELPFUL.
ONE IS THERE WAS A CLINICAL
TRIALS STEWARDSHIP TASK FORCE
THAT I PARTICIPATED IN, NOW A
YEAR AND A HALF OR SO AGO, THAT
LED TO RECOMMENDATIONS THAT
SUGGESTED SOME CENTRALIZATION OF
RESOURCES TO SUPPORT CLINICAL
TRIALS, PRIMARILY FOR SMALLER
INSTITUTES THAT CAN'T AFFORD TO
KIND OF CREATE ALL THIS
INFRASTRUCTURE IN HOUSE.
SO, WE'RE STILL EAGER TO SEE
WHAT OPPORTUNITIES DEVELOP IN
THAT SPACE THAT WE COULD
LEVERAGE.
ALSO FOR THOSE WHO HEARD MY
COUNCIL REPORT THIS MORNING, DR.
BERTAGNOLLI IS VERY INTERESTED
IN THE CONCEPT OF A PRIMARY CARE
RESEARCH NETWORK.
AND AT LEAST FOR SOME OF THE
CONDITIONS WITHIN OUR MISSION
AREAS, I COULD IMAGINE A FUTURE
IN WHICH WE CAN LEVERAGE THAT
CLINICAL TRIAL RESEARCH NETWORK
BECAUSE IT'S MEANT TO BE ALL
INCLUSIVE IN TERMS OF SERVICES
THAT WOULD BE PROVIDED.
I SEE KURT'S HAND UP.
>> ACTUALLY THE MOST ACTIONABLE
ACTUALLY IS THE PLANNING GRANT.
SO THE OTHERS ARE BIGGER, TAKE
LONGER.
I THINK THE COMMUNITY ENGAGEMENT
IS ACTUALLY THE BIGGEST CHANGE.
WE'VE HAD LISTENING SESSIONS
LIKE PLANNING, LONG-RANGE PLAN
OR STRATEGIC PLAN, WE'VE HAD
LISTENING SESSIONS FOCUSED ON
LARGE ADVOCACY GROUPS MOSTLY BUT
MEETING SEPARATELY, MUSCLE AS AN
INDIVIDUAL THING.
AND THOSE INFORMED US BUT WE
REALLY NEVER HAVE DONE THAT AS A
WAY OF DEVELOPING FUNDING
OPPORTUNITY ANNOUNCEMENTS FOR
CLINICAL TRIALS.
SO IMPLEMENTING THAT WOULD BE
THE SINGLE BIGGEST CHANGE.
THE ROLE OF COUNCIL WOULD BE THE
SECOND BIGGEST CHANGE.
BECAUSE, YOU KNOW, WE HAVEN'T
EFFECTIVELY DONE THAT EITHER,
CERTAINLY NOT IN ANY SYSTEMATIC
WAY ON LARGER CLINICAL TRIALS SO
THAT'S A BIG CHANGE.
BOTH WOULD BE EXPERIMENTS.
I'M NOT 100% SURE THAT WE WOULD
GET WELL-ROUNDED CONSENSUS
OPINIONS ABOUT THE DOMINANT
NEEDS IN A FIELD FROM A ONE-HOUR
LISTENING SESSION.
I DON'T KNOW.
WHETHER WHAT CAME OUT WOULD BE
THE BEST CLINICAL TRIAL TO FUND,
I DON'T KNOW.
THAT'S AN EXPERIMENT.
WHETHER THE INPUT OF COUNCIL --
YOU KNOW, COUNCIL DID GET
INVOLVED IN EVALUATION OF
LETTERS OF INTENT, FOR THE
GREATER THAN FIVE HUNDRED.
WE MET BEFORE EACH COWEN MEETING
MEET --
COUNCIL MEETING WITH EACH GROUP,
THEY NEVER DECLINED ANY OF THEM.
IT'S TOUGH.
WHETHER THIS SECOND LEVEL REVIEW
MAKING IT MORE RIGOROUS WILL
HAVE AN IMPACT, I DON'T KNOW.
I HOPE SO.
BUT, AGAIN, IT'S AN EXPERIMENT.
SO I THINK THE MOST ACTIONABLE
IS THE PLANNING GRANT.
AND INVOLVEMENT OF COMMUNITY AND
DEVELOPMENT OF SPECIFIC FUNDING
OPPORTUNITY ANNOUNCEMENTS AND
SECONDARY REVIEW OF COUNCIL,
THOSE ARE ALSO MAJOR CHANGES,
OUTCOME TBD.
GO AHEAD.
>> I JUST ALSO WANTED -- A LOT
HAS CHANGED FROM THOSE TIMES
WHEN COUNCIL APPROVED EVERY
LETTER OF RECOMMENDATION.
SO I THINK THAT ALSO WANTED TO
MAKE THE POINT THAT I THINK YOUR
WORKING GROUP HAS APPROPRIATELY
CALLED OUT CERTAIN AREAS WHERE
WE REALLY WOULD IDEALLY HAVE
MORE EXPERTISE WITHIN THE
INSTITUTE AND RECOGNIZE THAT AS
WELL.
THAT'S SOMETHING THAT SUPPORTED
BY YOUR RECOMMENDATIONS AND
EXPERIENCE OF THE CLINICAL
TRIALS WORKING GROUP WE'RE
WORKING TOWARDS SO THINK THAT'S
GOING TO BE AN IMPORTANT ONE OF
THE NEXT STEPS THAT WE DO.
THINK THAT WILL HELP US
TREMENDOUSLY.
>> NOT TO MENTION PATIENT
ENGAGEMENT, LEST YOU FORGET WE
WERE TALKING ABOUT TWO HOURS
AGO, THAT'S ONE OF THEM.
NO QUESTION, DIVERSITY IS
SOMETHING THAT WE'VE NOT FUNDED
STUDIES WITH PLANNED ENROLLMENT,
DID NOT THINK WAS SUFFICIENTLY
DIVERSE.
WE'RE NOT STARTING FROM GROUND
ZERO.
MAKING THAT MORE RIGOROUS IS A
GOOD WAY TO PUT IT.
MY DELIGHT HERE IS THAT THERE
ARE QUITE A FEW THINGS THAT WE
ACTUALLY CAN DO, AND I THINK
WILL DO, AND WE'LL SEE WHAT
IMPACT THEY HAVE.
SO, KURT?
>> I JUST WANTED TO REITERATE
WHAT JOAN SAID.
WE DID LOOK AT OTHER INSTITUTES
TO TAKE THE BEST OF THE BEST BUT
ALSO WITHIN THE CONSTRAINTS OF
THE FACT THAT FOR THE SIZE OF
OUR BUDGET AND AS THINGS THAT
ARE BROUGHT UP, THINGS THAT WE
PROBABLY BEST OF THE BEST TO
HAVE A CLINICAL TRIAL NETWORK,
BEST OF THE BEST TO HAVE A DATA
REPOSITORY, THOSE ARE NOT THINGS
THAT ARE WITHIN OUR BUDGET SCOPE
FOR NIAMS BUT THOSE ARE THINGS
WE HAVE TO LOOK FOR THE FUTURE.
SO WE DID TRY THE BEST OF THE
BEST, WITHIN REASON WITHIN OUR
CONSTRAINTS OF OUR INSTITUTE.
>> SUZANNE HAS A QUESTION.
>> I WONDER IF YOU WOULD
ELABORATE ON THE IMPACT OF
CLINICAL TRIALS AND HOW TO LOOK
AT THE QUESTION OF POTENTIAL
IMPACT IF IT'S CHANGING OF
CLINICAL PRACTICE, LONG-TERM
GOALS IN TERMS OF CHANGING
PRACTICE?
THAT WAS SOMETHING THAT WE HAVE
THOUGHT ABOUT IN THE PAST, VERY
CHALLENGING, I'M WONDERING IF
THERE ARE ANY INSIGHTS.
THE SECOND ONE IF YOU COULD
ELABORATE ON PARTNERSHIPS.
WE HAVE EXPERIENCED A STUDY AND
CLINICAL TRIALS SUPPORTED
THROUGH PARTNERSHIPS, IN
PARTNERSHIP WITH OTHER ENTITIES,
WONDERING IF THERE'S ADDITIONAL
CONSIDERATIONS THAT YOU WOULD
LIKE US TO THINK ABOUT.
>> YEAH, I AGREE, IMPACT IS A
TOUGH NUT TO CRACK.
YOU GAVE THE EXAMPLE DOES THIS
CLINICAL TRIAL HAVE THE
POTENTIAL TO CHANGE, AS YOU
SAID, CLINICAL PRACTICE OR
CHANGE TREATMENT GUIDELINES, SO
OUR PROFESSIONALS SOCIETIES
UPDATE TREATMENTS OF DISEASES,
IS THIS TRIAL GOING TO IMPACT
THOSE GUIDELINES WHICH WOULD
AFFECT EVERYBODY.
WOULD IT -- I GUESS WOULD IT
POSSIBLY PREVENT DISEASE OR
REDUCE INCIDENCE OF DISEASE?
OR REDUCE SIDE EFFECTS IN SOME
MANNER?
I GUESS ANYTHING THAT REALLY HAS
A DIRECT LINK, MAYBE SOMEONE
ELSE COULD DEFINE IT BETTER THAN
THAT.
IN TERMS OF PARTNERSHIP, MY OWN
PERSONAL EXPERIENCE, WE HAD TO
GO FIND PARTNERS AND WE WERE
CONFUSED HOW TO DO THAT.
WE SET UP MEETINGS, THE TRIAL,
WE MET WITH NIDDK, THEY SAID
WE'RE NOT INTERESTED.
MET WITH NHLBI, WELL, WE ONLY
FUND THIS TYPE OF TRIAL.
WE WENT TO ANOTHER INSTITUTE.
WE WERE UNCLEAR HOW TO GO ABOUT
THIS.
AND THEN CHUCK -- FINALLY --
SUZANNE, YOU WERE WORKING WITH
US CLOSELY ON THIS, AND WE THEN
WENT TO FNIH, THAT'S ULTIMATELY
HOW WE GOT PARTNERS FOR THAT
TRIAL, VERY SUCCESSFUL BUT CHUCK
TOLD ME DURING OUR MEETINGS THAT
HE'S VERY INVOLVED IF HE
SUSPECTS IN ADVANCE THAT
PROPOSED CLINICAL TRIAL WILL
REQUIRE ADDITIONAL FUNDING AND
HAS SEMBLANCE OF RESEMBLANCE TO
CARDIOLOGY OR NEPHROLOGY OR
SOMETHING LIKE THAT, HE WILL
START REACHING OUT IN ADVANCE TO
LOOK AT THE FEASIBILITY OF
FUNDING PARTNERSHIPS.
I GUESS "HEAL" IS A GREAT
EXAMPLE OF SOME PARTNERSHIPS AS
WELL.
OH I THINK JUST MAKING PEOPLE
AWARE THAT THEY DON'T HAVE TO
WANT TO PURSUE A CLINICAL TRIAL
IF IT'S TOO EXPENSIVE BUT CAN
TALK TO THE P.O. ABOUT THE
POSSIBILITIES OF PARTNERSHIPS
AND ADVISE AND HELP APPLICANT ON
THE PATH TOWARDS THAT IS WHAT I
WAS TALKING ABOUT.
>> JINOOS HAS A HAND UP AND I
WANT TO GIVE CHUCK THE
OPPORTUNITY, AND FOR OUR AD HOC
MEMBERS, PLEASE FEEL FREE TO
SPEAK UP IF YOU WANT TO SHARE
PERSPECTIVES.
FOR THOSE WHO MIGHT BE
CONTEMPLATING CLINICAL TRIALS,
WHAT CHALLENGES YOU SEE, AND
MAYBE TEO HAS A CONTINUE.
JINOOS, CHUCK, AND FROM THERE.
>> THANK YOU, JOAN.
JUST FANTASTIC.
THE BREADTH AND ALSO DEPTH OF
THE WORK YOU DID, IT'S VERY
IMPRESSIVE.
I'M EXCITED ABOUT NEW GRANT
MECHANISMS.
I JUST WONDER IF YOU COULD SAY A
LITTLE BIT MORE ABOUT THE
COUNCIL REVIEW, I WANT TO MAKE
SURE I UNDERSTAND SORT OF THE
BROADER PURPOSE.
DO YOU ENVISION A SITUATION
WHERE, FOR EXAMPLE, FIVE GRANTS
ARE REVIEWED BY COUNCIL BUT
THERE'S ONLY FUNDING FOR FOUR OF
THEM?
OR DO YOU ENVISION IT HAS
NOTHING TO DO WITH FUNDING AND
THERE'S DETERMINATION OF IMPACT?
WHAT'S LIKE THE GOAL OF THAT
COUNCIL REVIEW?
>> IT REALLY IS FOCUSED ON
IMPACT.
WE'RE NOT SAYING YOU SHOULD PUT
BUDGET INTO THE MOCKS.
I THINK -- INTO THE FIX, THAT'S
WHAT NIAMS FOLKS HAVE TO DO,
WEIGH THE BUDGET.
SOMETIMES TRIALS MAY BE RATED
HIGH, WE TALKED ABOUT THIS IN
OUR COMMITTEE, THAT TRIAL MIGHT
GET A VERY HIGH SCIENTIFIC
RATING FOR ITS FIELD, BUT WHEN
YOU THINK ABOUT WHAT IT'S GOING
TO CONTRIBUTE TO PATIENT CARE
AND QUALITY OF LIFE AND
SURVIVORSHIP AND WHATEVER ELSE,
IT'S KIND OF NOT -- IT'S
MIDDLING.
SOMETHING COULD HAVE A GREAT
SCIENTIFIC SCORE AMIDST THE
EXPERTS IN THE FIELD, BUT YET
NOT BE THAT IMPACTFUL.
THOSE ARE THINGS WE'RE TRYING TO
WEED OUT.
WHY FUND A TRIAL REGARDLESS OF
HOW STRUCTURALLY OR
SCIENTIFICALLY SOUND IT IS IF IT
DOESN'T SEEM TO HAVE IMPACT;
THAT'S REALLY THE EXTENT OF IT.
WE'VE ALL BEEN ON STUDY SECTIONS
WHERE YOU'RE THINKING, I DON'T
KNOW, THIS IS SO IMPORTANT BUT
ALL THE EXPERTS IN THAT FIELD
THINK IT'S SCIENTIFICALLY GREAT,
WE HAVE TO GO ALONG THOSE LINES,
SO THIS GIVES AN OPPORTUNITY TO
PUT IMPACT OUT THERE BY ITSELF.
>> MAYBE A FOLLOW-UP QUESTION
FOR LINDSEY, WE'RE TALKING ABOUT
SELECT PAY EARLIER TODAY.
ARE THERE CONNECTIONS BETWEEN
THESE TWO TOPICS?
>> YES, AND ALREADY APPLICATIONS
THAT -- IT'S CLINICAL TRIALS
THAT WILL BE ASSOCIATED WITH
BUDGETS, OVER $500,000 DIRECT
COSTS PER YEAR, NEEDS SPECIAL
PERMISSION TO APPLY, AND THEN
THE -- THOSE DECISIONS ARE NOT
PAYLINE DRIVEN.
WE HAVE A SELECT PAY PROCESS FOR
THOSE.
BACK TO THIS ISSUE, IT'S ABOUT
IMPACT, IT'S ALL ABOUT THE
DOLLARS WE HAVE, RIGHT?
YOU KNOW, AND BALANCING ACROSS
ALL OF THESE DIFFERENT AREAS.
BUT WE ALREADY, YOU KNOW, ARE
APPLYING THIS SELECT PAY PROCESS
TO ANY STUDY OVER A CERTAIN
DIRECT COST.
THERE'S ALSO, YOU KNOW, A
SEPARATE REVIEW PROCESS WE'RE
GOING TO GET TO LATER TODAY,
ASSOCIATED WITH INVESTIGATORS
THAT HAVE OVER A CERTAIN AMOUNT
OF FUNDING.
THAT'S KIND OF A DIFFERENT SET
OF ISSUES AND -- TO BALANCE, BUT
I AGREE, BUT ALSO MAYBE THIS IS
THE POINT YOU WERE GETTING AT.
JOAN GAVE A GREAT EXAMPLE OF WHY
WE THINK THE SELECT PAY PROCESS
IS IMPORTANT GENERALLY.
MEMBERS OF THE STUDY SECTION AT
CSR HAVE A CERTAIN PERSPECTIVE
AND THEY ARE CHARGED WITH A
CERTAIN SET OF CRITERIA THAT
THEY NEED TO ASSESS BUT IT IS
NOT CONSIDERING NECESSARILY
WHAT'S NEEDED MOST IN THE FIELD,
WHAT'S GOING TO HAVE THE MOST
IMPACT, WHAT ARE THE PRIORITIES
OF THE INSTITUTE THAT THINKS
ABOUT THOSE ISSUES EVERY DAY,
SO, YOU KNOW, WE CONSIDER OUR
JOBS TO BE TO CONSIDER THE
TECHNICAL MERITS SUCH AS
REFLECTED IN THE SCORE BUT TO
APPLY THIS OTHER FILTER, THIS
OTHER SET OF QUESTIONS, ABOUT
IMPACT.
AND SO WE WANTED TO HAVE
FLEXIBILITY TO BE ABLE TO FUND
SOME THINGS THAT ARE ABOVE THE
PAYLINE BECAUSE OF THESE OTHER
REASONS.
>> TEO, YOU WANTED TO MAKE A
COMMENT.
>> YES, THANK YOU.
I ALSO WANT TO THANK THE WORKING
GROUP FOR THE REALLY THOROUGH
AND THOUGHTFUL ASSESSMENT AND
EXCELLENT RECOMMENDATIONS.
I WANTED TO MAKE A COUPLE
COMMENTS.
ONE RELATES TO THE NOTION OF
IDENTIFYING THE MOST IMPORTANT
AND IMPACTFUL STUDIES TO BE
PURSUED.
I PARTICULARLY RESONATED WITH
THE COMMENT JOAN MADE, TO FOCUS
ON STUDIES THAT INDUSTRY WILL
NOT BE DOING BECAUSE WE WANT TO
COMPLEMENT THE APPROACHES BUT
ALSO FROM A PATIENT PERSPECTIVE
AND PATIENT ADVOCACY PERSPECTIVE
THE TYPES OF CLINICAL TRIALS
THAT AT LEAST WE HEAR FROM
PEOPLE LIVING WITH LUPUS THEY
ARE MOST INTERESTED IN HAVING
DONE AND PARTICIPATING IN
STUDIES LOOKING AT LIFESTYLE
INTERVENTION, HOW DOES THE DIET,
STRESS, IMPACT THE DISEASE
COURSE.
APPROACHES IN THEIR OWN LIVES,
SEEING GREAT SUCCESSES, AND SO I
HOPE THROUGH THIS STAKEHOLDER
ENGAGEMENT SOME OF THESE
APPROACHES OR CLINICAL TRIALS TO
STUDY THESE TYPES OF
INTERVENTIONS WILL COME FORWARD,
AND THAT LEADS ME TO MY
QUESTION.
WHETHER THIS ALSO PRESENTS AN
OPPORTUNITY, OR AN OPPORTUNITY
FOR US ACROSS CENTER AND
INSTITUTE DIRECTIONS THAT
LINDSEY IS A PROMOTING, MAYBE
WORKING WITH NATIONAL INSTITUTE
OF COMPLEMENTARY HEALTH,
WONDERING IF CONSIDERATIONS HAVE
BEEN MADE.
>> CERTAINLY YOU RAISE A GREAT
EXAMPLE OF HOW THERE MAY BE
CONSIDERATIONS SUCH AS IMPACT ON
PATIENT, PATIENT PERSPECTIVE
THAT WON'T NECESSARILY TRANSLATE
INTO GREAT SCORES BUT IF WE HAVE
A PROCESS FOR DETERMINING THEIR
IMPORTANCE THEN IT GIVES US AN
OPPORTUNITY TO BE DIRECTING
FUNDS IN THAT AREA.
IN TERMS OF PARTNERING WITH
INSTITUTES, WE JUST HAD A DEEP
DIVE DISCUSSION ABOUT
CO-FUNDING.
YOU KNOW, HOW CAN WE DEVELOP THE
MOST EFFECTIVE STRATEGY FOR
CO-FUNDING, MOST ADVANTAGEOUS IS
WHEN WE DIRECT INVESTIGATORS TO
OTHER OR WE LEVERAGE OTHER
RESOURCES THAT DON'T COME OUT OF
OUR BUDGET BUT THERE HAVE BEEN
SUCCESSFUL EXAMPLES OF
CO-FUNDING OR PARTNERING WITH
OTHER INSTITUTES.
IT'S MAYBE MORE CHALLENGING THAN
YOU'D LIKE BUT I THINK THE
COMMENT THAT WAS MADE ABOUT
CHUCK REACHING OUT EARLY,
SOMETIMES WE'RE IN THE BEST
POSITION TO KNOW WHAT OTHER
PARTNERS MIGHT EXIST ACROSS THE
NIH OR WHAT OTHER RESOURCE OR
FUNDING OPPORTUNITY, SO WE'RE
GENERALLY JUST BECAUSE OUR
BUDGET IS LIMITED THINKING ABOUT
THAT ALL OF THE TIME, TRYING TO
BE STRATEGIC.
SOMETHING WE'VE BEEN CHALLENGED
BY, TRYING TO UNDERSTAND, I'M
REMINDED OF AN EXAMPLE OF A VERY
EXPENSIVE TRIAL THAT WE WANTED
TO SUPPORT BUT WAS WELL, WELL
BEYOND OUR MEANS.
AND WE BASICALLY -- IT WAS
ADDITION WE SAID WE CAN PROVIDE
X, THE REST OF THE 80% OR
WHATEVER IT WAS YOU'LL HAVE TO
RAISE YOURSELF OR IDENTIFY.
THE INVESTIGATORS WERE ACTUALLY
ABLE TO DO IT.
AND BUT IT WAS KIND OF AWKWARD
SO DO WE COMMIT TO SOMETHING
WHERE WE ONLY HAVE 20% OF THE
BUDGET?
HOW DOES THAT PROCESS GET WORKED
OUT?
MORE IMPORTANTLY, JOAN OR
SOMEONE MENTIONED BEFORE, CAN WE
DEVELOP LIKE BEST PRACTICES FOR
HOW YOU IDENTIFY FUNDING
PARTNERS?
LET'S -- I MEAN, THE WORST CASE
AS
SCENARIO FOR INDIVIDUAL
INVESTIGATORS TO FIGURE OUT ON
THEIR OWN WHAT EXAMPLES CAN WE
REFINE AND SHARE TO HELP
EVERYBODY BUT IT'S COMPLICATED
TO DO THAT.
PLEASE.
>> TO FOLLOW UP ON THIS WITH, TO
COLLABORATIONS DON'T NECESSARILY
HAVE TO BE JUST THROUGH C.I.s,
MAYBE OUTSIDE ORGANIZATIONS,
MAYBE THEY ARE INTERESTED IN
PARTNERING FINANCIALLY OR WITH
IN-KIND ORGANIZATIONS, SOME HAVE
CLINICAL TRIAL NETWORKS.
JOAN MENTIONED WITH THE WORKING
GROUP RECOMMENDATIONS.
THERE MAY BE VALUE OTHER
PARTNERS COULD BRING TO THE
TABLE, PERHAPS NIAMS COALITION
MAY BE ONE APPROACH TO THAT.
>> AND THIS COMES UP, WE MEET
WITH ORGANIZATIONS REGULARLY
NOW, AND I THINK WE ALL SHARE
THAT GOAL BUT JUST HAVEN'T TAKEN
THE NEXT STEP.
LET'S DO A PILOT.
AND TRULY COMBINE OUR RESOURCES
IN WAYS THAT WORK FOR US, AND I
DON'T KNOW -- WE NEED TO FIGURE
OUT HOW TO DO THAT FOR SURE.
>> I THINK THE COMPLEMENTARY
HEALTH INSTITUTE, I FORGET THE
OFFICIAL NAME, THEY HAD THE SAME
ISSUE AND ENDED UP REVIEWING --
I FORGET THE MEDICAL TERM NOW,
REMOVING IRON FROM BLOOD, WEEKLY
FOR A YEAR, THEY ARE GETTING
LIKE A HUGE AMOUNT FROM NHLBI IN
PARTNERSHIP AND SOME FROM
ANOTHER INSTITUTE AND THEY HAD
TO PAY TO GO THROUGH THE SAME
PROCESS.
THEY ARE A VERY SMALL INSTITUTE.
I THINK THEY SAID WE HAVE TO
IDENTIFY THESE PROTOCOLS VERY
EARLY, THESE FUNDING
OPPORTUNITIES VERY EARLY BEFORE
WE EVEN REVIEW AND SEND IT FOR
REVIEW.
I THINK SMALL INSTITUTES OUGHT
TO TALK ABOUT THIS TOGETHER TOO.
SOME MORE PATHS MIGHT BE
DETERMINED.
>> RICARDO WOULD LIKE TO ASK A
QUESTION.
>> THANK YOU.
WONDERFUL WORK FROM THE WORKING
GROUP.
I HAVE A LITTLE CONCERN, FOCUS
ON RARE DISEASES, MY IMPRESSION,
I MIGHT BE WRONG, I DIDN'T FIND
ROOM FOR THAT KIND OF DISEASES,
I WAS THINKING ABOUT THE NIH
VERSUS THE INDUSTRY, THEY DOUBLE
OUR BUDGET OR MORE, RIGHT?
AND I KNOW THE INSTITUTE THAT
CAN MATCH SOME INDUSTRIES, NOT
OUR CASE, UNFORTUNATELY, SO
ANOTHER WAY TO THINK MAYBE IS TO
TRY TO DO THINGS THAT INDUSTRY
IS NOT DOING, ONE GOOD EXAMPLE
IS RARE DISEASES.
AND THE EXAMPLES OF DISEASE WITH
HUGE BURDEN EVEN IF NOT
PREVALENT AND I THINK THAT
SHOULD BE INCORPORATED SOMEHOW.
WE SHOULD NOT MIMIC INDUSTRY.
WE'LL NOT BE ABLE, AT THE NIAMS.
I WANT TO PUT THIS ON THE
TABLEs.
BUT THANK YOU SO MUCH FOR THE
DISCUSSION.
>> ANN?
WE HAVE A COUPLE MORE MINUTES.
AND THEN CHUCK.
ANN AND CHUCK.
>> I WONDERED, THIS WAS GREAT
WORK, ONE OF THE THINGS I
WONDERED ABOUT THOUGH, MAYBE I
MISSED IT IS, IF YOU HAVE
SOMEBODY BY SCORE THAT IS
FUNDABLE, BY PERCENTILE SCORES,
WILL THOSE BE LOOKED AT FOR
IMPACT AS WELL?
>> YES.
>> SO IT'S NOT JUST THE SELECT
PAY?
>> YES.
>> OKAY, GOOD.
>> CHUCK AND SHANTHINI.
>> LET HER ASK.
>> THANK YOU FOR BRINGING THIS
REALLY AMAZES WORK FORWARD.
I WANTED TO SHARE MY
PERSPECTIVE.
I'M LAUNCHING A PILOT STUDY
RIGHT NOW, A PILOT CLINICAL
TRIAL, AND I THINK ONE OF THE
CHALLENGES THAT I'M DEALING WITH
IS DIVERSITY BECAUSE I'M AT ONE
PLACE, AND SO EFFORTS TO SUPPORT
DIVERSITY AND RECRUITMENT,
WHETHER IT'S THROUGH A NETWORK
OR LEVERAGING THE CTSIs, WOULD
BE HELPFUL, MAYBE ESTABLISHED
INVESTIGATORS JUST STARTING OUT
IN CLINICAL TRIALS.
I WANTED TO EMPHASIZE THAT.
>> COMPLETELY AGREE.
THANK YOU SO MUCH.
CHUCK?
>> I GET THE LAST WORD.
I WANT TO THANK JOAN FOR THAT
GREAT PRESENTATION.
WHEN WE WORKED ON SLIDES, WOW,
WHAT IS SHE GOES TO SAY AFTER
SHE CUT THEM ALL DOWN, BUT THAT
WAS AMAZING.
I THANK EVERYBODY ELSE ON THE
WORK GROUP ON THIS CALL OR IN
THE ROOM.
AND ESPECIALLY BOB.
IT'S BEEN A LONG HAUL.
SEEMS LIKE WE STARTED YESTERDAY
AND FINISHED TODAY.
IT WAS A VERY GOOD GROUP.
ONE OF THE BETTER ONES I'VE EVER
BEEN ON.
I WILL MENTION TWO QUICK THINGS.
LOW-HANGING FRUIT, SO JUST FOR
PERSPECTIVE, THIS IS THE SAME
GROUP WHEN WE STARTED SAID GET
RID OF THE R 34.
BY THE TIME WE GOT TO THIS POINT
THEY SAID BRING IT BACK.
THAT'S HOW MANY DISCUSSIONS AND
WAYS -- IT'S HOW WE EVOLVED, WE
MADE RASH DECISIONS, CAME BACK
WITH A WAY -- IT'S NOT RIGHT.
I RESPECT THAT, WITH THE WAY
THEY MADE THEIR DECISIONS AND
RECOMMENDATIONS.
THE OTHER THING I'LL SAY WE JUST
PUBLISHED LAST WEEK, MAY 23,
UG3/UH3 NOFO.
A COUPLE THINGS NOT HIGHLIGHTED,
THERE'S NO CAP ON THE UH3, THE
THRILL -- TRIAL IS UNCAPPED.
SECOND WE TALKED ALREADY TODAY
AT LENGTH BEFORE LUNCH ABOUT
LIVED EXPERIENCE.
THAT IS NOW A REQUIREMENT FOR A
PLAN TO INCLUDE PATIENTS WITH
LIVED EXPERIENCE IN THE TRIAL,
IT'S GOING TO BE UP TO THE P.I.
HOW WE DECIDE TO INCORPORATE
THESE PEOPLE BUT IT'S A
REQUIREMENT THEY HAVE TO HAVE A
PLAN TO DO SO.
SO, EVERYTHING THAT THE WORK
GROUP HAS SAID, I'VE LISTENED
HARD TO AND WE'VE TRIED TO
INCORPORATE AS MANY AS WE CAN IN
NOFOs AND WILL CONTINUE TO DO
SO.
AGAIN, THANK YOU, JOAN, FOR A
GREAT PRESENTATION AND THANK YOU
TO THE WORK GROUP.
>> I LIKE ALL YOUR
RECOMMENDATIONS.
>> OKAY.
THANK YOU.
WE WILL BE CIRCLING BACK WILL
RARE DISEASE ISSUE TOO.
WHAT'S NEXT?
DARREN, PASSING TO YOU NOW.
>> CAN WE HAVE THE FIRST CONCEPT
CLEARANCE SLIDE?
OKAY, THANK YOU.
NEXT SLIDE.
LET ME -- BEFORE WE GET INTO THE
CONCEPT I WANTED TO MENTION WHAT
OUR PROCESS IS FOR CONCEPT
CLEARANCE, WHAT IT MEANS.
ESPECIALLY WE HAVE SOME NEW
FOLKS HERE.
THE WAY THE CONCEPT CLEARANCE
WORKS AT NIH AND WE IMPLEMENT IT
HERE AT NIAMS IS BEFORE WE COME
OUT WITH A LARGE FUNDING
OPPORTUNITY, CALL THEM NOFOs
NOW, WE NEED COUNCIL APPROVAL.
WE ALSO NEED AN OPTION FOR
PUBLIC COMMENT.
THAT'S WHAT WE'RE ASKING COUNCIL
FOR.
FOR TODAY'S CONCEPT CLEARANCE,
DR. MARY GARCIA-CAZARIN IS
INTRODUCE IT BROADLY SPEAKING.
WE'LL HAVE TIME FOR DISCUSSION
AND QUESTIONS BUT COMING BACK AT
SEPTEMBER COUNCIL NOT FOR
CONCEPT CLEARANCE BUT TO GET
YOUR INSIGHTS INTO MAYBE CHANGES
WE'RE GOING TO DO TO THE
PROGRAM.
SO I DIDN'T WANT YOU TO THINK
THIS WAS THE ONLY CHANCE YOU'RE
GOING TO HAVE TO WEIGH IN ON
THIS CONCEPT.
GOING FORWARD WE'LL TRY TO DO
THIS WITH ALL OF OUR CONCEPTS SO
WE CAN HAVE A FULLER DISCUSSION
AMONGST THE COUNCIL.
WITH THAT I'LL TURN IT OVER TO
MARY.
>> HELLO, GOOD AFTERNOON.
I'M HERE TO SEEK CONCEPT
CLEARANCE FOR THE THREE TYPES OF
RESOURCE CENTERS THAT THIS HAS
BEEN A LONGSTANDING PROGRAM OF
NIAMS, IN THE FOLLOWING AREAS.
RHEUMATIC DISEASE RESEARCH, SKIN
BIOLOGY AND DISEASES, AND BONE,
MUSCLE, ORTHOPEDIC RESEARCH.
CENTERS PROVIDED GREAT RESOURCES
FOR INVESTIGATORS.
I THINK ONE OF THE VERY
IMPORTANT OUTCOMES OF THE
RESOURCE CENTERS, TEAM SCIENCE,
I THINK THIS HAS ALLOWED FOR
COLLABORATIONS WITHIN
INSTITUTIONS AND DIFFERENT
DEPARTMENTS.
AND ALSO THIS HAS ALLOWED FOR
COLLABORATION ACROSS DIFFERENT
CENTERS AND OTHER INVESTIGATORS.
SO THAT IS A VERY IMPORTANT
HIGHLIGHT OF THE PROGRAM WE
WOULD LIKE TO CONTINUE.
THE OVERALL GOAL OF THE NIAMS
RESOURCE CENTERS IS TO PROMOTE
COLLABORATION INTERACTION AMONG
INVESTIGATORS THAT CONDUCT BASIC
SCIENCE OR CLINICAL SCIENCE,
CLINICAL RESEARCH.
WE ALSO -- ONE IMPORTANT THING
FOR RESOURCE CENTERS, WE HOPE
THAT THEY EXPAND THEIR OUTREACH,
AND ANOTHER THING WE DON'T WANT
TO DUPLICATE RESOURCES.
SO WE WANT TO MAKE SURE
INSTITUTIONS ARE PROVIDING
DIFFERENT SERVICES, SO THEREFORE
WE REALLY DON'T WANT TO HAVE
CENTERS THAT HAVE DUPLICATING
EFFORTS.
THE OVERALL OR GENERAL STRUCTURE
OF THE CENTERS HAVE INCLUDED
ADMINISTRATIVE CORE, SOME OF THE
CENTERS HAVE ONE OR MORE
RESOURCE CORES, AND ALSO THEY
HAVE HAD THE OPTION TO HAVE
PILOT AND FEASIBILITY PROJECTS.
THE PILOT AND FEASIBILITY
PROJECTS HAVE BEEN A GREAT
OPPORTUNITY TO FIELD SOME
SCIENTIFIC GAPS, INSTITUTIONS
ARE USING THESE PROJECTS TO
ACTUALLY BRING NEW INVESTIGATORS
INTO THE FIELD BY ALLOWING
POSTDOCS OR JUNIOR INVESTIGATORS
INTO PILOT AND FEASIBILITY
PROJECT.
EACH INSTITUTION DECIDES HOW TO
COMPETE FOR THE PILOT AND
FEASIBILITY PROJECTS, WE
CONSIDER THIS AN IMPORTANT PART
OF THE RESOURCE CENTERS.
SO I THINK THAT'S ALL I HAVE.
IN GENERAL WE'RE JUST SEEKING,
YOU KNOW, CLEARANCE FOR THESE
CONCEPTS FOR NIAMS TO CONTINUE
SPONSORING THESE RESOURCE
CENTERS.
>> THANK YOU.
BEFORE I CALL FOR A VOTE, I'LL
OPEN UP FOR DISCUSSION.
ANY QUESTIONS?
SURE, CHRIS.
>> HI, THANK YOU SO MUCH.
I APOLOGIZE, THIS IS A RATHER
IGNORANT QUESTION.
IS THIS KIND OF SIMILAR TO THE
P30 MECHANISM OR PERHAPS CAN
COMPARE AND CONTRAST WHAT THIS
CONCEPT IS DOING IF IT'S NOT
IDENTICAL TO THAT, IS IT A
SEPARATE ENTITY?
>> NO, IT IS A P30 MECHANISM.
>> OKAY, SORRY ABOUT THAT.
THANK YOU.
>> WE'D LOVE TO HEAR YOUR
COMMENTS.
>> YES, THIS HAS BEEN A P30.
WHAT WE'RE ASKING FOR PERMISSION
HERE IS TO LOOK AT THIS AND
CONSIDER REISSUING THIS OR
MAKING SOME CHANGES TO IT.
SO I GUESS THERE COULD BE A
POTENTIAL THAT WE MIGHT EXPAND
THE MECHANISM OR DO SOMETHING.
BUT THIS IS BASICALLY THE
RESOURCE CENTERS ARE THE P30s.
>> THAT'S VERY HELPFUL.
THANK YOU.
I'M A BIG FAN OF THEM.
>> YES.
>> SEVERAL OTHER COLLEAGUES OF
MINE ACROSS THE COUNTRY ARE
JUNIOR FACULTY AND LEVERAGED
RESOURCES OF P30s TO A GREAT
DEGREE.
>> ANY OTHER COMMENTS?
>> SEEING NONE, I'M GLAD TO HEAR
SOMEBODY REALLY APPRECIATES OUR
CURRENT P30s, I'LL MAKE A
MOTION WE APPROVE THIS CONCEPT
FOR FURTHER DEVELOPMENT BY THE
INSTITUTE.
DO I HAVE A SECOND?
SECOND, THANK YOU.
AND NOW I'LL CALL FOR ANYBODY
OPPOSED TO THIS MOTION, PLEASE
RAISE YOUR HAND OR SPEAK UP.
SEEING OR HEARING NONE, THIS
MOTION HAS BEEN APPROVED TO GO
FORWARD.
THANK YOU VERY MUCH, MARY.
>> IF THEY WANT TO SUBMIT
ADDITIONAL COMMENTS, THEY CAN DO
THAT, RIGHT?
>> YES, YOU CAN ALWAYS SUBMIT
ADDITIONAL COMMENTS AFTER
COUNCIL.
>> SO WE ARE AHEAD OF SCHEDULE.
WE'LL KEEP GOING AND COVER THE
FINAL SESSION OF THE OPEN
SESSION PRESENTED BY DR.
STEPHANIE BURROWS, CHIEF OF OUR
SCIENTIFIC PLANNING, POLICY, AND
ANALYSIS BRANCH TALKING ABOUT
OUR STRATEGIC PLAN, THE DRAFT
PLAN, I SAW THAT JANELLE DRUGIN
IS ONLINE, STEPHANIE WILL TALK
ABOUT THIS, WE'RE EAGER TO HEAR
AN UPDATE.
FOR THOSE THAT READ THE DRAFT
PLAN, HERE AND MOVING FORWARD WE
WELCOME AND ENCOURAGE YOUR
INPUT.
>> THANK YOU, DR. CRISWELL.
GOOD AFTERNOON.
AGAIN, I'M STEPHANIE BURROWS,
CHIEF OF OUR INSTITUTE
SCIENTIFIC POLICY PLANNING AND
ANALYSIS BRANCH.
AS WAS MENTIONED THIS MORNING
THE INSTITUTE IS IN THE PROCESS
OF DEVELOPING ITS STRATEGIC PLAN
FOR 2025-2029, AND MY BRANCH
ORGANIZES THIS PROCESS.
SO I'M DELIGHTED TO HAVE THE
OPPORTUNITY TO TELL YOU ABOUT
OUR PROGRESS TO DATE AND
HIGHLIGHT NEXT STEPS.
OKAY.
WHY SHOULD NIAMS HAVE A
STRATEGIC PLAN?
FIRST, CONGRESS REQUIRES NIH
INSTITUTES AND CENTERS TO HAVE A
STRATEGIC PLAN.
BUT AS IMPORTANT, THIS PLAN IS A
PUBLIC STATEMENT OF THE
INSTITUTE'S INTERESTS AND SINCE
IT'S THE FIRST PLAN WE'RE GOING
TO BE ISSUING UNDER DR.
CRISWELL'S LEADERSHIP WE IMAGINE
THE COMMUNITY WILL LOOK CLOSELY.
WE WANT TO MAKE SURE IT COVERS
OUR BROAD MISSION AND ALSO
REFLECTS THE INTERESTS OF OUR
RESEARCHERS AND COALITION
MEMBERS.
SO THE PLAN ALSO SERVES AS
INTERNAL REFERENCE FOR STAFF AT
ALL LEVELS AT THE INSTITUTE, ALL
COMPONENTS.
THIS SHOWS OVERVIEW OF THE STEPS
AND DEVELOPMENT OF THE STRATEGIC
PLAN.
I'M GOING TO ELABORATE ON SOME
OF THESE IN LATER SLIDES BUT THE
TAKEHOME MESSAGE IS WE'RE ON
TARGET TO HAVE YOU DISCUSS THE
FINAL VERSION OF THE PLAN AT THE
SEPTEMBER MEETING AND PUBLISH BY
END OF THE CALENDAR YEAR.
WE BEGAN IN 2022 WITH REQUEST
FOR INFORMATION, ASKING TWO
QUESTIONS.
ONE ABOUT CROSS-CUTTING THEMATIC
RESEARCH OPPORTUNITIES, THE
OTHER ABOUT BOLD ASPIRATIONS THE
INSTITUTE COULD PURCHASE SEE
OVER FIVE YEARS.
WE RECEIVED 46 RESPONSES, MOST
WERE FROM INDIVIDUALS AT
UNIVERSITIES OR HOSPITALS BUT
SOME WERE FROM PROFESSIONAL AND
VOLUNTARY ORGANIZATIONS.
SO REALIZING WE NEEDED TO HAVE A
MORE IN-DEPTH DISCUSSION OF SOME
IDEAS BUBBLING UP FOR
CROSS-CUTTING THEMES IN
SEPTEMBER OF LAST YEAR WE HELD A
THREE-DAY VIRTUAL IDEA
GENERATION MEETING.
AND THE IDEA HERE WAS TO CROWD
SOURCE ADDITIONAL IDEAS FOR
CROSS-CUTTING THEMES AND DISCUSS
THEM IN MORE DETAIL THAN WE
COULD THROUGH REQUEST FOR
INFORMATION.
SO WE BROUGHT TOGETHER
REPRESENTATIVES FROM ACROSS THE
INSTITUTE'S BROAD INTERESTS TO
INTERACT AND TO BUILD ON ONE
ANOTHER'S IDEAS.
SO, PARTICIPANTS, SOME ARE
PICTURED HERE, INCLUDED
RESEARCHERS FROM VARIOUS
BACKGROUNDS AND CAREER STAGES,
NIAMS COUNCIL MEMBERS,
REPRESENTATIVES OF PROFESSIONAL
AND VOLUNTARY ORGANIZATIONS AS
WELL AS REPRESENTATIVES FROM
OTHER FEDERAL AGENCIES AND NIH
INSTITUTES, CENTERS AND OFFICES.
I KNOW SEVERAL OF YOU WERE
INVOLVED IN THIS MEETING, AND WE
GREATLY APPRECIATE YOUR
PARTICIPATION.
SO, AFTER THAT MEETING WE TOOK
THE IDEAS THAT HAD EMERGED AS
WELL AS INFORMATION WE HAD
GOTTEN FROM THE FIRST REQUEST
FOR INFORMATION AND CONSOLIDATED
INTO NINE THEMES.
WITH CORRESPONDING BOLD
ASPIRATIONS.
WE ISSUED A SECOND REQUEST OPEN
FOR SIX WEEKS AND RECEIVED MORE
THAN TWO DOZEN RESPONSES, NINE
FROM COALITION MEMBER
ORGANIZATIONS.
WE GOT SOME ADDITIONAL
SUGGESTIONS FOR CROSS-CUTTING
THEMES OR BOLD ASPIRATIONS BUT
MOSTLY RESPONDENTS WERE FOCUSED
ON FINE TUNING THE IDEAS THAT WE
PRESENTED IN THAT RFI.
OKAY.
SO, THE PRIORITIES THAT ARE
GOING INTO THE STRATEGIC PLAN
REFLECT CURRENT VIEWS OF THE
PATIENT AND RESEARCH COMMUNITY
TODAY, BUT WE RECOGNIZE SCIENCE
CHANGES QUICKLY.
SO THE INSTITUTE NEEDS TO REMAIN
RECEPTIVE TO FUNDING THE MOST
PROMISING APPLICATIONS EVEN IF
TOPICS MOVED ON BEYOND THE
TOPICS OR PRIORITIES WE'RE
ARTICULATING IN STRATEGIC PLAN.
THEREFORE, WE'RE ENCOURAGING TO
THINK ABOUT NEEDS RELATED TO
FOUR GOALS.
A BRIDGED ON THE SLIDE, THE
FIRST IS ADVANCING THERAPY
DEVELOPMENT FOR EVERYONE BY
IDENTIFYING HUMAN BIOLOGICAL AND
BEHAVIORAL AND ENVIRONMENTAL
FACTORS THAT AFFECT HEALTH,
DISEASE, AND THE PATIENT
EXPERIENCE.
AND TESTING INTERVENTIONS IN
PEOPLE WITH ARTHRITIS AND
MUSCULOSKELETAL AND SKIN
DISEASES.
SECOND FOSTERING DISCOVERIES TO
IMPROVE HUMAN HEALTH BY
DEVELOPING RESOURCES, INCLUDING
RELEVANT PRE-CLINICAL MODELS,
UNBIASED CLINICAL DATA AND
COMPUTATIONAL BIOLOGY
APPROACHES.
THIRD, SUPPORTING NECESSARY
INFRASTRUCTURE AND ROBUST
DIVERSE RESEARCH WORKFORCE
CAPABLE OF CONDUCTING ALL TYPES
OF STUDIES INCLUDING
INTERDISCIPLINARY RESEARCH AND
TEAM SCIENCE.
AND FINALLY COMMUNICATING TIMELY
AND RELEVANT ADVANCES TO
PATIENTS, COMMUNITIES, AND
SCIENTISTS.
SO THESE GOALS ARE INTENDED TO
REMAIN CURRENT AND OVERARCHING,
AGAIN EVEN IF THE TOPICS THAT
WE'RE FUNDING MOVE BEYOND
PRIORITIES THAT ARE INCLUDED IN
THE PLAN.
LET'S MOVE TO WHAT THOSE
PRIORITIES ARE.
SO, BASED ON WHAT WE HEARD FROM
THE COMMUNITY ABOUT THE STATE OF
THE SCIENCE AND NEEDS AND
OPPORTUNITIES NOW WE'VE
ARTICULATED SIX RESEARCH
PRIORITIES.
THEY ARE ABRIDGED ON SLIDES SO
I'LL READ THE FULL TEXT FOR YOU.
FIRST, STUDYING MOLECULAR AND
CELLULAR MECHANISMS OF HEALTH
AND DISEASE IN HUMANS AND
UNDERSTANDING RESULTS PHENOTYPES
AND ENDOTYPES.
DEVELOPING REGENERATIVE MEDICINE
APPROACHES, USING DATA SCIENCE,
ARTIFICIAL INTELLIGENCE, AND
MACHINE LEARNING AND
COMPUTATIONAL BIOLOGY TO ADVANCE
KNOWLEDGE AND TREATMENTS.
UNDERSTANDING BEHAVIORS AND
ENVIRONMENTAL EXPOSURES THAT
PROMOTE HEALTH OR CAUSE DISEASE.
TRANSLATING FINDINGS INTO
INTERVENTIONS THROUGH CLINICAL
AND EPIDEMIOLOGIC STUDIES.
FINALLY, CONDUCTING RESEARCH ON
HEALTH DISPARITIES AND IMPROVING
HEALTH EQUITY, ALL APPLY TO ALL
FIVE DISEASE AND TISSUE-SPECIFIC
MISSION AREAS.
WE'LL BE MONITORING PROGRESS
ACROSS THE BOARD TO UNDERSTAND
HOW WE CAN ACCELERATE ADVANCES.
SOME OF THE NEEDS AND
OPPORTUNITIES THAT WE HEARD AND
ONE OF THE ASPECTS OF OUR
MISSION REALLY REQUIRE
MECHANISMS THAT GO BEYOND
TRADITIONAL R01 RESEARCH PROJECT
GRANT.
WE'VE ARTICULATED THOSE AS WHAT
WE ARE CALLING CAPACITY BUILDING
PRIORITIES AND THERE'S ONE
OPERATIONAL PRIORITY.
AND SO THE THREE CAPACITY
BUILDING PRIORITIES ARE
DEVELOPING ENABLING RESEARCH
INFRASTRUCTURE INCLUDING
TECHNOLOGIES AND NOVEL SYSTEMS.
SUPPORTING INTERDISCIPLINARY
RESEARCH TEAM SCIENCE AND
PARTNERSHIPS, AND PROMOTING
TRAINING AND ROBUST DIVERSE
WORKFORCE.
WE ARTICULATED OUR MISSION
ASPIRATIONAL THEME, A PRIORITY,
CONDUCTING INFORMATION
DISSEMINATION AND ENGAGEMENT
ACTIVITIES.
YOU HAVE BEFORE YOU TODAY THE
MAIN SUBSTANTIVE COMPONENTS OF
OUR STRATEGIC PLAN.
YOU HAVE MOST OF THE
INTRODUCTORY MATERIAL.
YOU HAVE THE TEXT OF THE FOUR
GOALS AND TEN PRIORITIES THAT I
WALKED YOU THROUGH.
AND THEN COUNCIL SHOULD HAVE
RECEIVED IN THE ELECTRONIC
COUNCIL BOOK IN ADVANCE THE FULL
TEXT WHICH ALSO INCLUDES
EXAMPLES OF RELEVANT ACTIVITIES
THAT COULD RELATE TO EACH
PRIORITY AS WELL AS RESOURCE
BOXES THAT DETAIL RELATED
RESOURCES THAT COULD BENEFIT THE
COMMUNITY.
SO, WHEN YOU SEE THE THIRD
REQUEST FOR INFORMATION WHICH
WE'RE GOING TO ISSUE THIS SUMMER
AS WELL AS FULL STRATEGIC PLAN,
FINAL DRAFT OF THE PLAN, IN
SEPTEMBER, THERE ARE GOING TO BE
TWO NOT INCLUDED NOW.
FIRST WILL INCLUDE EXAMPLES OF
RESEARCH ACCOMPLISHMENTS FROM FY
2020 THROUGH FY 2024 THAT RELATE
TO THE STRATEGIC PLAN AND THEN
LAST WILL BE AN APPENDIX ON HOW
WE INTEND TO -- IT'S AN OVERVIEW
HOW WE INTEND TO IMPLEMENT AND
REPORT ON OUR PLAN.
I'M JUST GOING TO CLOSE WITH THE
NEXT STEPS.
SO AFTER TODAY WE'RE GOING TO
INCORPORATE YOUR COMMENTS AND
THEN WE'LL ISSUE A THIRD REQUEST
FOR INFORMATION SO THE COMMUNITY
CAN HAVE INPUT ON THE NEAR FINAL
PLAN.
SO VERY IMPORTANTLY, IF YOU HAVE
ADDITIONAL FEEDBACK AFTER THIS
MEETING, WE WOULD LIKE YOUR
WRITTEN COMMENTS BY TUESDAY,
JUNE 4.
SO WE INTEND TO HAVE A FINAL
DRAFT OF THE PLAN FOR YOU TO
REVIEW BEFORE THE SEPTEMBER
COUNCIL MEETING, AND THEN
PUBLISH -- THEN AFTER VARIOUS
ADMINISTRATIVE CLEARANCES WE'LL
PUBLISH THE PLAN BY THE END OF
THE CALENDAR YEAR.
SO WITH THAT OVERVIEW I WANT TO
TURN THE MEETING BACK OVER TO
DR. CRISWELL TO LEAD THE
DISCUSSION TO COLLECT YOUR
COMMENTS.
THANK YOU VERY MUCH.
>> GREAT.
THANK YOU.
I WANT TO THANK YOU AND YOUR
TEAM AND THE MANY FOLKS, PROGRAM
OFFICERS, LEADERSHIP, WHO WORKED
SO HARD TO GET US TO THIS POINT.
IT REALLY DOES REFLECT A
TREMENDOUS AMOUNT OF INPUT FROM
THE COMMUNITY, THE BROADER
COMMUNITY.
SO EAGER TO HEAR ANY THOUGHTS,
REACTIONS, SUGGESTIONS,
COMMENTS, ANYBODY WOULD LIKE TO
SHARE NOW, AND WE'LL BE LOOKING
OUT FOR COMMENTS AND SUGGESTIONS
MOVING FORWARD AS YOU HEARD
THROUGH NEXT TUESDAY AND AFTER
THE LAST RFI.
I SEE JONELLE DRUGAN, DID YOU
WANT TO ADD ADDITIONAL COMMENTS
SINCE YOU'VE BEEN SO HEAVILY
INVOLVED?
>> I WANT TO ECHO YOUR
APPRECIATION TO MY COLLEAGUES AS
WELL AS TO THE COMMUNITY.
WE REALLY FELT GOING INTO THIS
UNDER DR. CRISWELL'S LEADERSHIP
THAT THIS NEEDED TO BE COMMUNITY
DRIVEN.
WE NEEDED TO HEAR FROM THE
COMMUNITY WHAT THE NEEDS AND
OPPORTUNITIES WERE.
AND THEN TAKE THAT INFORMATION
AND DISTILL IT INTO SOMETHING
THAT WE THOUGHT COULD POSITION
THE FIELD TO MOVE FORWARD.
SO, THANK YOU TO ALL WHO HAVE
PARTICIPATED AND ALL WHO ARE
GOING TO PARTICIPATE AS PART OF
THE COUNCIL PROVIDING INPUT.
THANK YOU.
>> ANN?
>> I HAVEN'T SEEN -- HAVEN'T
BEEN ABLE TO READ, THERE'S A
MORE STRATEGIC PLAN?
>> YOU DID NOT GET THE LINK TO
THE e-COUNCIL BOOK?
WE'LL MAKE SURE WE GET THAT TO
YOU.
>> IS THERE -- IS IT A FULL
DRAFT STRATEGIC PLAN AT THIS
POINT OR JUST THE BULLETS HERE?
>> FULL DRAFT.
>> OKAY, HERE.
>> MICROPHONE.
>> SO THERE ARE A COUPLE
APPENDICES WE'RE STILL WORKING
ON BUT OTHERWISE IT'S THE FULL
DRAFT.
>> OKAY.
I WONDER IF MAYBE THERE COULD BE
A LITTLE BIT LONGER THAN FIVE
DAYS FOR THE COUNCIL TO TAKE A
FULL LOOK NOW THAT WE'LL HAVE
HAD A LITTLE DISCUSSION MAYBE
EXTEND ANOTHER WEEK OR SOMETHING
JUST TO MAKE SURE THAT YOU GET
IT.
MY SECOND QUESTION IS, ONE OF
OUR BULLETS SAID THERE WOULD BE
PUBLIC COMMENT BETWEEN JULY AND
SEPTEMBER.
IS THAT THE PLAN AS WELL?
>> YES.
>> BECAUSE I KNOW ORGANIZATIONS
WILL WANT TO WEIGH IN AS WELL AS
RESEARCHERS.
>> ABSOLUTELY.
WE'LL POST THE FULL DRAFT PLAN
ON THE WEB.
ONCE WE'VE INCORPORATED
COUNCIL'S COMMENTS, WE'LL POST
FOR PUBLIC COMMENT ONCE AGAIN.
>> WONDERFUL.
LOOK FORWARD TO THAT.
THANK YOU.
>> YOU ALL HAD THE OPPORTUNITY
TO FIRST WEIGH IN ON THIS DRAFT
AND THEN WE'LL TAKE BACK YOUR
COMMENTS.
AND AN UPDATED DRAFT FOR THE
BROADER COMMUNITY.
>> THANK YOU.
THANK YOU FOR ALL YOUR WORK ON
THIS.
I WENT THROUGH THE GRUELING
SESSIONS IN SEPTEMBER.
>> GRUELING?
THEY WERE FUN!
THEY WERE FUN.
>> SOME OF THEM ESPECIALLY.
>> I SAW I THINK MARIA AND I
THINK VALENTINA?
>> I COULDN'T FIND THE DRAFT
EITHER.
WHEN I PRESS ON THE LINK UNDER
YOUR NAME IT TAKES ME TO THE
COMMON AREA LOGGING PAGE, NOT A
DOCUMENT.
I DIDN'T REALIZE THERE WAS
SOMETHING TO READ BECAUSE UNDER
THERE THERE WASN'T, AT LEAST FOR
ME, THERE'S NO DRAFT UNDER POINT
SEVEN.
I DON'T KNOW IF IT'S ME ONLY.
BUT IT TAKES ME TO A LOG-IN PAGE
FOR THE COMMENTS PAGE.
>> FOR ME IT WORKS.
YOU GUYS GOT IT, OKAY.
>> BEFORE YOU GO LET'S MAKE SURE
WE GET YOU ACCESS, THOSE WHO
DIDN'T GET SENT.
>> WE CAN ALSO E-MAIL THE
DOCUMENT TO YOU TOO.
>> YEAH.
>> THAT MIGHT BE EASIER IF
YOU'RE HAVING ACCESS ISSUES.
>> YEAH, IT DOWNLOADED BUT FOR
ME, I DON'T KNOW IF IT'S BECAUSE
I HAVE A MAC.
>> IT'S A MAC ISSUE.
>> YOU'LL SEND IT OUT TO
EVERYBODY BY E-MAIL, TO MAKE
SURE YOU ALL HAVE IT.
>> WE'LL E-MAIL IT.
>> YEAH.
>> THIS IS JEANNE.
I'M EAGER TO HEAR ABOUT THE
IMPLEMENTATION PLAN.
IT'S A WORK IN PROGRESSES,
RIGHT?
I CAN'T WAIT.
>> IT'S CUED UP FOR STEPHANIE TO
SEND TO YOUR LEADERSHIP TEAM.
WE WANTED TO HEAR COUNCIL'S
COMMENTS IN CASE THEY SAID WE
HATED IT AND WANT YOU TO REDO
THE WHOLE PLAN.
IT'S CUED UP, ASSUMING THAT WE
AREN'T REWRITING THE PLAN BACK
AT STEP ZERO IT WILL BE IN YOUR
IN-BOX AFTER THIS COUNCIL
MEETING.
>> GREAT.
>> DR. JEPSON HAS A HAND UP?
>> YEAH, I DON'T KNOW IF -- SO
PROVOCATIVE MY COUNCIL MEETING
BUT I'M GOING TO ANYWAY.
I READ THROUGH THE SUMMARY OF
THE STRATEGIC PLAN, REFLECTED
THREE DAYS VERY WELL.
ONE THING I WAS HOPING TO SEE A
LITTLE BIT MORE OF WAS EMPHASIS
ON PRO-ACTIVE PERSONALIZED
TREATMENTS.
THIS WOULD INCLUDE DIAGNOSTIC
TREATMENTS BECAUSE ONE THING IN
THE MUSCULOSKELETAL WORLD THAT
WE'RE DOING OKAY WITH BUT NOT
REALLY SPECTACULARLY WITH IS
GETTING IN FRONT OF DISEASE
CONDITIONS RATHER THAN ALWAYS
BEHIND THEM.
WE ALWAYS SEEM TO BE IN
RESTORATIVE TREATMENT PLAN.
WE MAY -- YOU KNOW, WE MAY BE
PUSHING OURSELVES, OUR
COMMUNITY, OUR FIELD, TO REALLY
EMPHASIZE RESEARCH THAT PUSHING
THAT ENVELOPE.
I'LL PUT COMMENTS IN THERE WHEN
THAT TIME COMES UP BUT I WAS --
MAYBE OTHER PEOPLE, COUNCIL
MEMBERS, HAD THOUGHTS ON THAT
TOO.
>> THANK YOU.
YOU REMINDED ME THERE'S ALSO A
SECTION ON BOLD ASPIRATIONS THAT
YOU DIDN'T MENTION BUT THAT ALSO
COULD BE A PLACE WHERE THAT SORT
OF VISIONARY THINKING,
ASPIRATIONAL GOALS, COULD BE
REFLECTED.
THANK YOU AND PLEASE SEND IN
COMMENTS.
WE'RE TAKING NOTES BUT CAPTURE
THOSE.
DR. BOTSWAY HAS A HAND UP TOO.
>> THANK YOU FOR THE
PRESENTATION AND I'M ALSO -- I
SUFFERED ISSUES AND MAY BE
SPEAKING OUT OF TURN BUT WANTED
TO ASK TWO MAYBE CLARIFYING
QUESTIONS IN TERMS OF THINKING.
FIRST RELATES TO THE BULLET IN
RELATIONSHIP TO THE HEALTH NEEDS
OF DIVERSE POPULATIONS,
HISTORICAL UNDERREPRESENTED
GROUPS.
MORE SPECIFICALLY HOW IS NIAMS
OR IS THAT IDEA INCLUSIVE OF
EMERGING INSIGHTS INTO THE ROLE
OF LIKE SOCIAL DETERMINANTS OF
HEALTH AND CONCEPTS AND
ALLOSTATIC LOAD, CHRONIC
STRESSORS IN CREATING
DISPARITIES, DISEASE BURDEN,
TREATMENT EFFICACY, DOES THAT
ENCOMPASS THOSE IDEAS AND HOW IS
IT COMMUNICATED TO THE PUBLIC?
>> YEAH, THANK YOU FOR THAT
COMMENT.
MY COLLEAGUES MIGHT WANT TO ADD
TO THIS.
ABSOLUTELY.
AND KIND OF ON THAT NOTE, ONE OF
THE THINGS WE HEARD MORE NOW
THAN I THINK WAS REFLECTED IN
THE PREVIOUS PLAN WAS
RECOGNITION OF THE ENVIRONMENTAL
CONTRIBUTION TO DISEASE VERY
BROADLY DEFINED INCLUDING
CERTAINLY SOCIAL DETERMINANTS OF
HEALTH, CLIMATE CHANGE, ET
CETERA, AND THIS I THINK
ILLUSTRATES ONE OF MANY VALUES
OF THE STRATEGIC PLAN IS WE
DON'T HAVE A LOT OF RESEARCH IN
THAT AREA SO THIS CAN HELP US
IDENTIFY GAPS AND OPPORTUNITIES.
BUT ABSOLUTELY THAT WOULD BE
PART OF THAT CONCEPT, AND
JONELLE WERE YOU GOING TO
RESPOND TO THIS QUESTION TOO?
>> I WAS.
>> GO AHEAD.
>> TO READ A COUPLE BULLETS, ALL
IN YOUR ELECTRONIC COUNCIL BOOK,
IDENTIFY THE BIOLOGIC SOCIAL AND
ENVIRONMENTAL CAUSES OF
INIQUITIABLE HEALTH OUTCOMES, IN
PEOPLE FROM UNDERREPRESENTED
GROUPS WHO HAVE DISEASES
ADDRESSED BY NIAMS.
DETERMINING HOW UNDERREPRESENTED
GROUPS CAN BENEFIT FROM PROVEN
INTERVENTIONS THROUGH COMMUNITY
ENGAGEMENT AND IMPLEMENTATION
SCIENCE EFFORTS.
AND THOSE ARE TWO EXAMPLES.
AND THEN WE ALSO HAVE SOME
BULLETS ON IMPROVING DIVERSITY
OF RESEARCH PARTICIPANTS.
>> CONCEPTS RELATED TO
ALLOSTATIC LOAD ARE IMPORTANT,
SOCIAL DETERMINANTS OF HEALTH
CAME ACROSS CLEARLY THERE.
ONE OTHER COMMENT IN
RELATIONSHIP TO THE REGENERATIVE
MEDICINE, BASED ON MY OWN
EXPERIENCE DOING SICKLE CELL
DISEASE RESEARCH.
I MAY BE MISSTATING, IT'S BEEN
MY EXPRESSION SUBMITTED
PROPOSALS THROUGH EXTRAMURAL
PROGRAM SICKLE CELL RESEARCH
SOMEHOW HAS TO GO TO NHLBI OR
SOMETHING LIKE THAT, THERE'S
SOME TYPE OF A RULE ABOUT WHICH
INSTITUTES ARE ASSIGNED SICKLE
CELL DEGREES GRANTS, NHLBI
DOESN'T DO R21, IT DOESN'T GO
ANYWHERE, AS ONE OF THE
CONSEQUENCES.
AND SO I ASK THAT IN
RELATIONSHIP TO REGENERATIVE
MEDICINE, AND WHETHER IT IS
INCLUSIVE OF IDEAS AND
RELATIONSHIP TO AGING, I DON'T
KNOW WHETHER THERE'S ANY KIND OF
STRICTURES ABOUT THE DEGREE TO
WHICH AGING FOCUSED RESEARCH HAS
TO GO THROUGH NIA AS A PRIMARY
INSTITUTE OR CAN -- OR IS NIAMS
UNFETTERED IN ITS ABILITY TO
SUPPORT AGING AS A COMPONENT OF
WE ARE KIND OF THOSE FUTURE
INITIATIVES IN THE REGENERATIVE
MEDICINE SPACE?
I FEEL LIKE THIS TOPIC IS
GAINING A LOT OF WELL-DESERVED
APPRECIATION AND IT'S IMPORTANT
TO REGENERATIVE MEDICINE
THERAPY, THERAPEUTIC
DEVELOPMENT, I WANTED TO ASK IN
TERMS OF THE STRATEGIC PLAN, YOU
KNOW, WHETHER THERE MAY BE ANY
SIMILAR TYPES OF RESTRICTIONS OR
COMPLEXITY IN HOW THAT MAY BE
SUPPORTED.
>> YEAH, ONE OF THE THINGS
YOU'RE REFERRING TO HAS TO DO
WITH REFERRAL, I'M BLANKING ON
THE NAME, REFERRAL GUIDANCE, A
PROCESS OF REFERRAL THAT
DETERMINES WHERE GRANTS GET
DISTRIBUTED, TO WHAT INSTITUTES
AND CENTERS.
IT GETS QUITE SPECIFIC.
THERE ARE ALSO A LOT OF GRAY
AREAS.
THAT'S ONE ISSUE.
IT'S NOT NECESSARILY -- FOR
EXAMPLE, ABSOLUTELY NIAMS CAN
FUND SOME PROJECTS THAT RELATE
TO AGING.
THEY DON'T HAVE TO ALL GO TO
NIA.
IT DEPENDS ON OVERALL SCOPE.
ALL THAT COULD BE ONE REASON FOR
HAVING SECONDARY -- RECOMMENDING
SECONDARY INSTITUTES, IF IT GOES
INITIALLY TO NIA AND THEY ARE
NOT PARTICULARLY INTERESTED, IT
WAS A TOPIC WE WERE VERY
INTERESTED IN, THE ISSUE OF THE
R21 GRANTS HAS BEEN CHALLENGING
FOR US BECAUSE -- IN FACT THAT'S
WHY WE CHANGED OUR POLICY ABOUT
R21, BECAUSE WE WERE RECEIVING
R21 GRANTS OUTSIDE OF OUR
MISSION AREAS BECAUSE VARIOUS
INSTITUTES AND CENTERS HAD
STOPPED FUNDING THOSE.
THAT'S AN ISSUE THAT WE'VE
ADDRESSED PRETTY WELL NOW.
BUT I DON'T KNOW WHETHER ANY
COLLEAGUES THINK ABOUT THIS ALL
THE TIME, REFERRAL GUIDELINES
WANT TO ADD, GAYLE?
>> YEAH, ONE THING YOU RUN INTO
IS THE FACT THAT A LOT OF
INSTITUTES DON'T PARTICIPATE IN
THE R21.
AND IN THOSE CASES, THEY
WOULD -- THE REFERRAL FOLKS AT
CSR WOULD LOOK FOR THE NEXT
APPROPRIATE INSTITUTE, SO YOU
CAN TRY TO TARGET WITH YOUR
LANGUAGE WHEN YOU WRITE UP OUR
INTRODUCTION AND MAYBE BROADEN
THE SCOPE OF WHAT COULD BE
APPLICABLE FOR YOUR APPLICATION
TO BE ASSIGNED TO, INSTITUTES IT
COULD BE APPROPRIATE.
SO, THOSE ARE THE KIND OF THINGS
YOU MIGHT WANT TO TALK ABOUT
WITH EITHER AARON MARKOWITZ IN
NIAMS OR TALK WITH NHLBI PROGRAM
DIRECTORS ABOUT HOW TO BE SURE
THAT THIS FINDS A HOME.
A LOT OF TIMES WE JUST DON'T GET
THESE.
SO THEY ARE NOT SENT TO US AS AN
OPTION.
>> MY UNDERSTANDING, ALL GRANTS
HAVE TO GO SOMEWHERE, RIGHT?
THAT'S WHERE SOME CHALLENGES --
YEAH.
I KNOW DR. HARRIS-TRYON HAS A
HAND UP.
WAS ANYBODY ELSE GOING TO
RESPOND TO THIS CURRENT POINT
BEFORE WE MOVE TO -- OKAY.
IN THAT CASE, PLEASE, NEXT
QUESTION.
>> THIS IS TAMIA.
IN A SIMILAR VAIN, I'M SO
EXCITED TO SEE BEHAVIOR AND
ENVIRONMENT INCLUDED AS SOMEONE
WHO STUDIES THE SKIN MICROBIOME,
IT CANNOT BE UNTANGLED, IT'S
GREAT TO SEE THE INITIATIVES IN
THE STRATEGIC PLAN.
>> SINCE YOU ARE MENTIONED THIS
AGAIN ONE OF THE THINGS THAT
STRUCK ME FOLLOWING THAT IDEA
GENERATION MEETING IS TWO THEMES
THAT REALLY STOOD OUT TO ME,
AMONG MANY OTHERS, WAS MORE
INTERESTED EMPHASIS ON THE
ENVIRONMENT AND MORE INTEREST
AND CONCERN ABOUT
INFRASTRUCTURE.
AND SAYING INSTITUTES DO
EVERYTHING TO SUPPORT
INFRASTRUCTURE SO WE DON'T HAVE
INDIVIDUAL INVESTIGATORS AND
INSTITUTIONS TRYING TO RECREATE
EVERYTHING ON THEIR OWN.
WE HEARD THAT CALL LOUD AND
CLEAR.
OTHER -- YES, TEO.
>> THANK YOU, YOU'VE DONE A
REALLY TREMENDOUS JOB PULLING
THIS FEEDBACK TOGETHER INTO THIS
DRAFT PLAN.
MY QUESTION WAS THE DISCUSSIONS
EARLIER THIS MORNING ON PATIENT
ENGAGEMENT AND RECOMMENDATIONS
ON CHANGES TO THE CLINICAL
TRIALS SUPPORTED THROUGH NIAMS,
IS THAT GOING TO BE INTEGRATED
INTO THE STRATEGIC PLAN OR WILL
THAT BE DONE IN PARALLEL ONCE
THE STRATEGIC PLAN IS BEING
IMPLEMENTED?
>> SO, CERTAINLY WE WILL TAKE --
INCORPORATE THE FEEDBACK WE
RECEIVED TODAY FROM COUNCIL AND
YOU'LL NOTICE A SECTION, WE LEFT
A PLACE HOLDER TO DESCRIBE SOME
OPPORTUNITIES UNTIL AFTER THIS
MEETING.
YES, WE CONSIDER THIS TO BE PART
OF WHAT'S INFORMING WHAT WILL BE
INCLUDED IN THE PLAN.
>> OTHER THOUGHTS THIS?
RECOGNIZE THAT MOST OF YOU
HAVEN'T SEEN THIS DRAFT.
ANY OTHER THINGS YOU DIDN'T
HEAR, THINGS YOU WANT CLARIFIED?
COMMENTS FROM OTHERS ON THE
NIAMS TEAM IN TERMS OF THE
PROCESS OR ISSUES THAT YOU
THOUGHT WERE VERY PROMINENT?
YEAH, JONELLE?
>> SO SINCE WE DEFINITELY WANT
COUNCIL'S COMMENTS I'M GOING TO
HAVE TO WORK WITH OUR
COMMUNICATIONS AND COPY EDITOR
FOLKS ON HOW TO GET THEIR
FEEDBACK BUT WE DEFINITELY,
SINCE SO MANY OF YOU DIDN'T GET
THE VERSE TO REVIEW, WE
DEFINITELY NEED TO GIVE YOU
ANOTHER WEEK.
I'M GOING TO TAKE THE
INITIATIVE, INSTEAD OF WAITING
TO TALK TO STEPHANIE ABOUT IT
JUST SAY INSTEAD OF THE 4th
HAVE ANOTHER WEEK AND DO IT THE
12th AND WE'LL FIGURE OUT HOW
TO GET IT REVIEWED SO WE'RE ON
OUR DEADLINE.
WE THOUGHT WE GAVE YOU PLENTY OF
TIME.
IF YOU CAN'T OPEN THE FILE, YOU
CAN'T OPEN THE FILE.
>> YOU SHOULD ALL HAVE JUST
RECEIVED AN E-MAIL FROM SHOLANDA
WITH THE DOCUMENTS.
YOU HAVE IT AS OF NOW AND
JONELLE WILL BE COUNTING DOWN
YOUR TIME.
>> I'LL START RIGHT NOW.
>> I'M JUST REMINDED THAT I JUST
WANT TO ACKNOWLEDGE ONE OF OUR
GUESTS TODAY, AMY PAYNE, DR. AMY
PAYNE, WHO HAS HELPED THE
INSTITUTE IN MANY WAYS INCLUDING
MOST RECENTLY AS THE CHAIR OF
OUR BOARD OF SCIENTIFIC
COUNSELORS AND, AMY, IF YOU'RE
NOT IN A PLACE TO TURN ON YOUR
CAMERA, NO PRESSURE.
THANK YOU SO MUCH FOR JOINING
US.
YOU HAVE HELPED THE INSTITUTES
IN SO MANY WAYS.
AND REALLY APPRECIATE YOUR
INTEREST.
YOU SHOULD FEEL FREE TO SPEAK UP
ABOUT -- I DON'T KNOW IF YOU SAW
THE DRAFT PLAN OR NOT OR JUST IF
YOU HAVE ANY COMMENTS NOW OR
MOVING FORWARD THAT WOULD BE
GREAT.
>> ABSOLUTELY.
THANK YOU SO MUCH.
I'M TECHNICALLY AN OBSERVER, WHY
I WAS KEEPING MY CAMERA OFF.
IT'S BEEN WONDERFUL WORKING WITH
NIAMS.
THANK YOU FOR THE OPPORTUNITY TO
SERVE ON THE COUNCIL GOING
FORWARD.
I THOUGHT THE DRAFT PLAN CAN A
GREAT JOB TRYING TO ADDRESS
CROSS-CUTTING INITIATIVES, TO
INTEGRATE THE BONE BIOLOGY
RHEUMATIC AND SKIN DISEASE
COMMUNITY.
I THINK THOSE ARE REALLY GREAT
OPPORTUNITIES I LIKE HOW YOU
INVESTED IN INFRASTRUCTURE AND
TRAINEES, AN AREA OF OPPORTUNITY
AND THREAT, WE HAVE ALL THESE
EXCITING THERAPIES THAT ARE
POTENTIALLY COMING TO TRIALS AND
MANY SPECIALTIES SAYING WE DON'T
HAVE CLINICAL INVESTIGATORS TO
RUN THESE TRIALS TO DO THAT.
I THINK ONE THING THAT WE
THOUGHT ABOUT WAS WHETHER OR NOT
THERE MIGHT BE OPPORTUNITIES TO
ALMOST CO-INVEST IN SOME
TRAINING OPPORTUNITIES FOR
CLINICAL INVESTIGATORS AND EVEN
TRANSLATIONAL INVESTIGATORS
BETWEEN GOVERNMENT, MAYBE
THROUGH A K/INDIVIDUAL F AWARD
AND INDUSTRY TO CO-SPONSOR
BECAUSE A LOT OF INDUSTRY WILL
IDENTIFY THEY OFFER INTERNAL
POSTDOCTORAL PROGRAMS, AT THE
SAME TIME RECOGNIZE PERHAPS THEY
DON'T HAVE THE STRUCTURES TO
FULLY SUPPORT THAT TYPE OF
TRAINING.
SO THAT MIGHT BE A REALLY GOOD
AREA OF PARTNERSHIP.
AND THEN THE ONLY OTHER PIECE OF
FEEDBACK I HEARD AS WELL WAS TO
MAKE SURE WE'RE NOT TOO
DIRECTIVE ON THE HUMAN RELEVANCE
BECAUSE A LOT OF THINGS THAT WE
LEARN COME FROM UNEXPECTED AREAS
TO OPEN IT UP THAT A LOT OF OF
OUR LEARNING CAN COME FROM MODEL
SYSTEMS, COMPARING AND
CONTRASTING COMPARATIVE
IMMUNOLOGY, COMPARATIVE BIOLOGY,
ET CETERA, SO JUST KEEPING OPEN,
AN OPEN FRAMEWORK FOR JUST
DISCOVERY IN GENERAL TO MOVE
FORWARD SCIENCE.
>> THANK YOU FOR THOSE COMMENTS.
FEEL FREE TO SEND IN WRITTEN
COMMENTS IF YOU THINK WE HAVE AN
OPPORTUNITY TO HIGHLIGHT THAT
MORE SIGNIFICANTLY.
SO THANK YOU SO MUCH.
ANY OTHER COMMENTS AT THIS
POINT?
OKAY.
I'M LOOKING AT THE AGENDA.
YEAH, WE HAVE UNTIL 3:00.
WE'LL CLOSE THAT PART OF OUR
MEETING BUT IF IN THE REMAINING
MINUTES OTHER IDEAS COME UP WITH
THE STRATEGIC PLAN PLEASE FEEL
FREE.
WE CAN CIRCLE BACK.
WE HAVE SOME TIME NOW.
AS MUCH AS YOU'D LIKE, AT LEAST
UNTIL 3:00, BUT NOT NECESSARILY
UNTIL 3:00.
IT'S NOT NECESSARY FOR YOU TO
CIRCLE BACK TO PREVIOUS TOPICS
DURING THE MEETING, NEW ISSUES,
THINGS YOU'D LIKE TO HEAR ABOUT,
THINGS WE CAN PREPARE AND HELP
YOU UNDERSTAND ACTIVITIES AT THE
NIH, ET CETERA.
VALENTINA?
>> YEAH, TO CIRCLE BACK TO THE
CLINICAL TRIAL AND DISCUSSION WE
HAD, WHETHER THE COUNCIL SHOULD
DISCUSS CERTAIN CLINICAL TRIAL,
EVALUATE IMPACT.
MAYBE WANTED TO OPEN MORE
REFLECTION FIRST ON THE
COMPOSITION OF THE COUNCIL, TO
CLINICAL IMPACT TO PATIENTS,
WHAT DOES IT MEAN FOR US TO HAVE
TO RECRUIT COMPETENCE TO MAKE
REFLECTION ON THAT, WILL WE
SHIFT COMPOSITION, OR BASED ON
PRINCIPLE, WHETHER COUNCIL WAS
THE RIGHT DECISION, WHAT ARE WE
GOING TO DO IN ORDER TO BE
THOUGHTFUL ABOUT IT, AND SECOND
GENERALLY IN THE STRATEGIC
PLANNING IN GENERAL WOULD LOVE
TO ECHO WHAT AIMEE WAS SAYING,
CONTINUE TO HAVE AN INVESTMENT
ON, YOU KNOW, RESEARCH R01
BASED, A LOT OF RESOURCES
BECAUSE WE NEVER KNOW WHERE
INNOVATION COMES FROM.
AND BY CLOSING AND CREATING MORE
SELECTIVE INVESTMENT ON CERTAIN
THINGS THAT APPLY WILL REMOVE
BASIC RESEARCH IN ORDER FOR US
TO KNOW TWO YEARS FROM NOW,
COUNTER ACT THE NEXT PANDEMIC
AND SUCH.
>> REGARDING THE FIRST ISSUE,
ABOUT THE COMPOSITION AND,
QUOTE, COMPETENCE OF COUNCIL AND
BOB WANTS TO TAKE THAT QUESTION.
AS SOME OF YOU MAY BE AWARE,
THERE HAS BEEN AN UNFORTUNATE
BACKLOG FOR A NUMBER OF YEARS IN
COUNCIL SLATE APPROVALS.
BUT THE DEPARTMENT IS CATCHING
UP AND IN FACT SO WE HAVE DR.
JEPSEN AND SEVEN PEOPLE IN THE
QUEUE WHO WE HOPE WILL JOIN US
IN SEPTEMBER.
THE COUNCIL IS SMALLER THAN EVER
RIGHT NOW.
THAT DOESN'T NECESSARILY MEAN
WE'RE ALWAYS GOING TO HAVE -- AN
WE GIVE A LOT OF THOUGHT AND
THERE'S A LOT OF OVERSIGHT WITH
RESPECT TO REPRESENTATIVENESS OF
THE COUNCIL, COMPOSITION IN ALL
RESPECTS, GEOGRAPHY, BACKGROUND,
SCIENTIFIC AREA, ET CETERA.
SO THAT DOESN'T NECESSARILY
SATISFY WHAT YOUR POINT IS BUT
BOB YOU WANTED TO SPEAK TO THAT
FIRST.
>> THERE'S AN ISSUE WITH A
LITTLE TWIST ON WHERE YOU PUT
THINGS.
YOU KNOW, THE WAY JOAN EXPRESSED
IT, EXPERTISE IN STUDY SECTION,
STUDY SECTION RECRUITS AD HOC
MEMBERS TO HAVE THE EXPERTISE TO
COVER.
YOU MAY HAVE NOTICED THE
SIDEBAR, YOU SIT DOWN ON STUDY
SECTION, YOU HEAR THE PEOPLE WHO
REALLY ARE CLOSE TO THE FIELD
SAYING, YEAH, WE GOT TO DO THIS.
BUT YOU KNOW THE AREA.
FOR ME LET'S SAY RHEUMATOLOGY
BUT MAYBE NOT VASCULITIS, AS A
RHEUMATOLOGIST, THAT MAY BE
IMPORTANT TO PEOPLE WHO FOCUSED
ON THAT FIELD BUT IN THE BIGGER
SCHEME MAYBE NOT.
AND THAT'S WHERE WE'RE LOOKING
FOR COUNCIL INPUT.
IT'S NOT AT THAT REALLY GRANULAR
LEVEL OF EXPERTISE.
COUNCIL IS MADE UP OF PRETTY
SENIOR PEOPLE WITH AD HOCS TO
BRING IN THE JUNIOR -- WHATEVER
THE RIGHT WORD IS, PERSPECTIVE.
BUT THE COUNCIL MEMBERS ARE
REQUIRED TO HAVE PRETTY BROAD
EXPERTISE.
AND WHAT WE'RE DOING IS TAKING
ADVANTAGE OF THAT MORE THAN THE
REALLY FINE BOOK EXPERTISE.
>> ANN?
>> THOUGHTS ON COUNCIL'S ROLE ON
IMPACT, I WOULD THINK, I WOULD
HOPE, THAT STAFF WOULD GIVE SOME
RECOMMENDATIONS AND SOME
CONTEXT.
>> THAT'S PART OF IT.
>> SO WHAT HAPPENS CURRENTLY FOR
CLINICAL TRIALS AND OTHER
APPLICATIONS THAT ARE REVIEWED
INTERNALLY IS OUR INTERNAL STUDY
SECTIONS SCORE THEM, THEY ARE
DISCUSSED, WITH LEADERSHIP, ET
CETERA, AND THE PROGRAM
OFFICERS, RELEVANT PROGRAM
OFFICERS, WRITE OUT A
RECOMMENDATION, CRITIQUE.
AND THEN WE DISCUSS IT.
SO, WE'RE REALLY TALKING ABOUT
INCLUDING COUNCIL MEMBERS, A
SUBSET OF COUNCIL MEMBERS IN AT
THAT POINT.
AND, YOU KNOW, WE CONSIDER THE
SCORE BUT WE CONSIDER A LOT OF
OTHER THINGS.
AND WE COUNT ON THE PROGRAM
OFFICER TO HAVE DUG DEEPER.
OFTEN THEY WERE IN REVIE
COMMITTEE MEETING, THEY HAVE
COMMUNICATED WITH THE
INVESTIGATOR, THEY KNOW THE
FIELD VERY WELL.
SO, I GUESS I'M INTERESTED IN
HEARING FROM YOU, YOU ALL FEEL
DIFFERENTLY, IS THIS A ROLE, NOT
SPECIFICALLY DEFINED YET, THAT
HE WOULD EMBRACE AND FEEL
COMFORTABLE WITH, YOU KNOW, SO
WHAT WE'RE TALKING ABOUT IS
HELPING WEIGH IN, BEING ANOTHER
BODY, TO WEIGH IN ON THE VERY
DIFFICULT DECISIONS WE HAVE TO
MAKE, WHICH CLINICAL TRIALS TO
FUND, WE HAVE SCORES ASSIGNED TO
ALL OF THEM.
BASED ON IMPACT, BUDGET IS
IMPORTANT TOO.
REALLY, WE HAVE TO FACTOR THAT
IN.
WE'RE NOT ASKING YOU TO MAKE THE
FINAL DECISION BUT IS THAT AN
ACTIVITY THAT YOU THINK YOU
COULD CONTRIBUTE PRODUCTIVELY
TO?
THE WORKING GROUP ARE PROPOSING
THAT, DOESN'T MEAN WE HAVE TO DO
IT.
WE CAN THINK CREATIVELY ABOUT
HOW TO IMPLEMENT THAT.
JANOOS ALREADY ASKED ABOUT THIS
SO SHE'S BEEN THINKING ABOUT.
WHAT DO YOU ALL THINK?
>> FIRST I WANT TO SAY QUICKLY
--
>> OH, ED HAD A HAND UP.
DID YOU WANT TO WEIGH IN ON THIS
POINT?
>> NO, A DIFFERENT POINT.
IT CAN WAIT.
>> ALL RIGHT.
>> THANK YOU FOR SAYING BUDGET
MATTERS.
I FEEL LIKE WE DON'T HEAR THAT
ENOUGH.
IT'S SOMETHING THAT REALLY DOES
MATTER.
THIS MAY SOUND A LITTLE BIT OFF
TO SAY BUT WHAT'S -- I THINK
PHYSICIAN-SCIENTISTS, PHYSICIANS
ARE IN A GOOD POSITION TO
COMMENT.
WE HAVE SOME PhD scientists.
BUT MISSING CAN PEOPLE WITH
INDUSTRY EXPERIENCE.
WE HAVE TO DEAL WITH CONFLICT OF
INTEREST ISSUES BUT OFTEN HAVE
PEOPLE WHO HAVE INDUSTRY
EXPERIENCE WHO MIGHT BE WELL
POSITIONED TO COMMENT ON SOME OF
THESE THINGS BECAUSE WHERE NIAMS
AND NIH IN GENERAL CAN BE MOST
STRATEGIC IS, AGAIN, THOSE
THINGS THAT INDUSTRY, THEY ARE
NOT GOING TO PURSUE BUT IT MIGHT
BE REALLY IMPORTANT TO LOOK AT
THIS CURRENTLY GENERICALLY
AVAILABLE DRUG WE WANT TO KEEP
THEM FROM PATENTING, ET CETERA,
NICE OPPORTUNITIES ESPECIALLY
RHEUMATOLOGICAL DISEASES, WE'VE
SEEN THAT GO WRONG.
SO I THINK WE MIGHT WANT TO LOOK
AT SOME PEOPLE WHO HAVE FORMER
INDUSTRY EXPERIENCE THAT COULD
BE HELPFUL TO SOME OF THIS
DECISION MAKING.
>> OTHER OPINIONS, REACTIONS TO
THIS POINT?
GAYLE?
>> I WAS JUST GOING TO SAY IN
SOME CASES, YOU KNOW, COUNCIL
MEMBERS ARE AT MAJOR RESEARCH
INSTITUTIONS.
YOU MAY HAVE COLLEAGUES OR
FRIENDS WHO ARE ACTIVELY
ENROLLING OR CARRYING OUT
CLINICAL TRIALS, AND PEOPLE WHO
ARE EXPERTS IN CLINICAL
MEDICINE, SO GETTING SOME INPUT
FROM THEM, BRINGING THAT BACK TO
THE COUNCIL IS ALSO VERY
APPROPRIATE.
I WAS THINKING ABOUT PEOPLE WHO
WERE AT HARVARD OR BETH ISRAEL
OR SOME OF THE DIFFERENT
INSTITUTIONS IN BOSTON THAT
THERE ARE PEOPLE DOING BRILLIANT
STUDIES.
AND FINDING OUT WHAT THE IMPACT
OF A PARTICULAR APPROACH WOULD
BE OR PARTICULAR PROPOSAL,
WITHOUT ACTUALLY SHARING THE
PROPOSAL ITSELF BUT JUST GETTING
THE GESTALT FOR WHAT THIS GROUP
OR INDIVIDUAL MIGHT THING WOULD
BE IMPORTANT FOR US.
YOU REPRESENT WITH WHAT YOU
BRING TO THE TABLE THE ENTIRETY
OF WHAT YOU'RE EXPOSED TO.
>> OTHER OPINIONS FROM MEMBERS
OF COUNCIL WHETHER THIS IS
SOMETHING THEY THINK WOULD BE A
GOOD ROLE FOR COUNCIL OR, YOU
KNOW, IN WHAT -- YOU KNOW, HOW
BEST -- JANOOS, DID YOU WANT TO
SPEAK TO THIS OR A SEPARATE
ISSUE?
>> I WAS GOING TO SAY THAT I
THINK IT'S WORTH A PILOT, YOU
KNOW, WITHOUT MAKING A LONG-TERM
COMMITMENT.
I THINK WE NEED TO SORT OF WORK
OUT THE ISSUES, LIKE EXACTLY
WHAT THE GOAL IS, EXACTLY WHAT
SORT OF THE RATING SYSTEM IS
GOING TO BE.
AND I IMAGINE THIS IS REALLY
GOING TO BE SORT OF AN ADVISORY
ROLE TO HELP NIAMS STAFF MAKE A
DECISION WITHOUT GETTING TOO
MUCH INTO THE WEEDS OF THE
METHOD.
WHAT WE DON'T WANT IS FOR THIS
TO TURN INTO ANOTHER STUDY
SECTION, FOR PEOPLE TO NITPICK.
>> COMPLETELY AGREE.
>> ANYWAY, FOR ALL THOSE REASONS
I THINK I WOULD BE INTERESTED IN
JUST TRYING IT ONCE, SEEING IF
IT ACTUALLY HELPS.
THE ULTIMATE GOING IS TO HELP
NIAMS MAKE GOOD DECISIONS, SO IF
IT'S HELPFUL LET'S CONTINUE.
IF IT'S NOT THAT HELPFUL IT
WOULD BE FINE TO DITCH IT TOO.
>> THAT'S GREAT.
SOUNDS LIKE GREAT ADVICE.
I SEE A LOT OF HEADS NODDING
AROUND THE ROOM.
SO TO BE CONTINUED, OBVIOUSLY.
ED, DO YOU WANT TO GO NEXT?
YOUR HAND HAS BEEN UP A WHILE.
>> SURE.
SINCE WE HAVE SOME TIME, I JUST
WANTED TO ECHO A COMMENT,
FORGIVE ME, I DON'T KNOW WHO IT
WAS FROM, MADE EARLIER,
REGARDING MAINTAINING THE
EMPHASIS ON RARE DISEASE AND
IMPORTANCE OF RARE DISEASE
RESEARCH.
THESE ARE CONDITIONS THAT OFTEN
LACK SUFFICIENT ATTENTION IN
FUNDING BUT THEY REALLY HOLD
KEYS TO UNLOCKING SOME IMPORTANT
BIOMEDICAL INSIGHT.
I WANT TO URGE NIAMS TO CONTINUE
PRIORITIZING AND EXPANDING ITS
EFFORTS IN RARE DISEASE AS WELL,
AND JUST TO ENSURE THAT I THINK
SOME OF THE GREAT STEPS BEING
TAKEN NOW TO THINK STRATEGICALLY
ABOUT THE FUTURE AND MAXIMIZE
IMPACT WITH THE INVESTMENTS THAT
NIAMS IS MAKING THAT WE REALLY
LEVERAGE EVERY OPPORTUNITY TO
MAKE SIGNIFICANT STRIDES FOR
THOSE THAT ARE AFFECTED BY THOSE
CONDITIONS.
>> THANK YOU.
YOU KNOW, I THINK IT'S
APPROPRIATE TO SAY OR TRUTHFUL
TO SAY THAT NIAMS HAS A
REPUTATION OF BEING SUPPORTIVE
MUCH RARE DISEASE RESEARCH.
I'VE HEARD FROM SO MANY PEOPLE,
I'M CONVINCED OF THAT.
AND SOME OF YOU MAY HAVE HEARD
FROM SUSANNA ABOUT THE BESPOKE
GENE THERAPY CONSORTIUM AND WE
WERE THRILLED TWO INITIAL
CLINICAL TRIALS UNDERTAKEN IN
THAT EFFORT WERE RARE DISEASES
WITHIN OUR MISSION AREAS.
I DON'T KNOW IF PETER COX IS
BEHIND ME BUT WE AND A COUPLE
OTHERS WERE AT THE SKIN -- I'M
BLANKING, SOCIETY OF
DERMATOLOGY, I THOUGHT THE S WAS
SKIN, SOCIETY FOR INVESTIGATIVE
DETERMINE NOTHING, AND WE HEARD
A LOT ABOUT THIS.
PETER STEPPED UP TO THE TABLE.
PLEASE FEEL FREE CHIME IN.
>> GOING BACK TO THE COUNCIL
ISSUE, FOR ME IN THE PAST, IT'S
ALWAYS BEEN A FIREWALL BETWEEN
SCIENTISTS WHO DO REVIEW OF
GRANT PROPOSALS AND INSTITUTION
THAT MAKES FINANCIAL DECISIONS.
SO IT LOOKS TO ME A LITTLE BIT
WE'RE REMOVING THAT FILE.
IF THAT'S GOOD FOR THE P.I.s,
I DON'T KNOW.
IT WILL NOT BE GOOD FOR YOU
BECAUSE YOU WILL BE TARGETED AS
THE ONES THAT INFLUENCE FUNDING
DECISION, NOT REVIEW.
I THINK IT'S A POINT AT LEAST TO
THINK ABOUT.
>> PETER, THAT'S ACTUALLY NOT
RIGHT.
COUNCIL HAS ALWAYS BEEN SECOND
LEVEL OF REVIEW.
>> FOR ALL APPLICATIONS.
>> FOR ALL APPLICATIONS IN
STATUTE.
ALL WE'RE DOING IS PUTTING SOME
MEAT ON THOSE BONES.
>> I WAS WONDERING WILL THE
GRANULARITY, HOW MUCH THEY WILL
BE INVOLVED.
>> WE ARE WAY OUT ON ONE EXTREME
ON LIMITATIONS OF INVOLVEMENT OF
COUNCIL IN SPECIFIC FUNDING
APPLICATIONS.
THOSE ARE THE ONCE WE'RE
STATUTORILY REQUIRED TO BRING TO
COUNCIL.
SO, THIS -- YES, SO WE -- THIS
IS NOT PUTTING COUNCIL AT RISK
COMPARED TO ANY OTHER COUNCIL
ANYWAY.
>> JUST TO CLARIFY THAT POINT,
SO BECAUSE WE'VE THOUGHT ABOUT
THIS A LOT, WHAT SHOULD BE
APPROPRIATE ROLE OF COUNCIL.
WHEN I CAME ON BOARD I HEARD
MEMBERS FELT THEY WEREN'T
ENGAGED, THEY WOULD COME AND
HEAR THINGS.
WE'VE BEEN THINKING HOW BEST TO
PRODUCTIVELY WORK WITH COUNCIL
WITHOUT OVERBURDENING THEM.
SOME COUNCILS GO THROUGH EVERY
APPLICATION, LIKE JINOOS WAS
SAYING, IN MY MIND KIND OF YOUR
WORST NIGHTMARE.
SO YOU HAVE STUDY SECTION.
AND THEN HAVE YOU NIAMS STAFF.
THEN IT'S LIKE STARTING ALL OVER
AGAIN WITH COUNCIL.
SO WE HAVE BEEN VERY RELUCTANT
TO FOLLOW THAT MODEL AND
WE'VE -- WE'RE ON THE OTHER END
OF THE SPECTRUM BUT WE'RE OPEN
TO ADOPTING AND ADJUSTING OUR
PROCEDURES IN WAYS THAT ARE
MEANINGFUL TO YOU, HELPFUL TO
THE INSTITUTE, ET CETERA.
SO --
>> I THINK JINOOS HAD IT RIGHT,
FOR US COUNCIL WAS ADVISORY,
LOOKED TO YOU FOR ADVICE.
EVERYTHING WE DO.
YOUR ADVICE IS SOUGHT, WELCOMED,
ACTUALLY NEEDED.
WHAT ONE AREA WE'VE NOT SOUGHT
IT SO MUCH IS INDIVIDUAL
DECISIONS.
BUT I THINK JINOOS EXPECTED THE
BALANCE QUITE WELL THAT I THINK
IT'S RIGHT THAT IF A COUNCIL
SAYS NO, ACTUALLY VOTES
SOMETHING DOWN, MAJORITY OF THE
COUNCIL SAYING DON'T FUND IT, AT
LEAST I'M NOT SURE IF IT'S
STATUTORY BUT A LOT OF US WILL
NOT FUND SOMETHING UNDER THOSE
CONDITIONS.
I'M NOT SURE IF IT'S BUT LAW
IT'S ADVISORY, HELPING US MAKE
BETTER DECISIONS.
JINOOS HAD THE BALANCE RIGHT.
>> ED, WE DIDN'T -- WE'RE NOT
FULLY ADDRESSING YOUR POINT.
I THOUGHT PETER WAS GOING TO
TALK ABOUT THAT AND I TALKED
ABOUT SOMETHING TOTALLY
DIFFERENT.
I WAS THINKING ABOUT OUR
EXCHANGE AT THE SID MEETING.
>> RARE.
>> YES, RARE DISEASES AND THAT'S
EXHIBITED BEAUTIFULLY IN OUR
INTRAMURAL RESEARCH PROGRAM TOO
SO WE'RE VERY INTERESTED IN RARE
DISEASE FOR MANY REASONS, THEY
CAN TEACH US ABOUT OTHER FORMS
OF DISEASE.
GETTING -- CIRCLING BACK TO
CLINICAL TRIALS PORTFOLIO, I
THINK JOAN CLARIFIED THIS.
A BALANCE.
NOT AT THE EXCLUSION BUT A
BALANCE.
>> IT'S TRUE WE'VE REALLY VALUED
SOME PARTICULAR STUDIES IN RARE
DISEASES.
TWO REASONS.
A LOT OF THOSE ARE MENDELIAN,
YOU KNOW, MONOGENIC AND YOU CAN
LEARN ABOUT HUMAN BIOLOGY THAT
YOU CANNOT LEARN FROM MICE, FROM
STUDYING MONOGENIC CHANGES IN
HUMANS.
THE OTHER IS THAT TREATMENTS
THAT TARGET VERY SPECIFIC
PATHWAYS WILL OFTEN WORK IN
MONOGENIC DISEASES IN WAYS THAT
ARE ACTUALLY REALLY AMAZING.
AS SHOWN BY WORK DONE IN RFP,
INCLUDING JOHN JAY'S WORK,
JAK/STAT INHIBITORS,
INTERFERONOPATHIES OR
PARANOPATHIES, DRUGS THAT TARGET
SPECIFIC PATHWAYS ARE MIRACLES.
THE WAY JOHN PUT IT, 10% CHANGE
IN A POPULATION WITH
OSTEOARTHRITIS IS ACTUALLY
REMARKABLE BENEFIT AT THE
SOCIETY LEVEL BUT 50% CHANGE IN
A PATIENT WITH A RARE DISEASE
CAN BE EQUALLY IMPACTFUL.
IN PART BECAUSE OF EVERYTHING
ELSE YOU LEARN AS A RESULT OF
THOSE STUDIES.
SO I'M OBVIOUSLY NOT A LIFER,
BUT AS LONG AS I'VE BEEN HERE
THE INSTITUTE HAS ALWAYS THOUGHT
THAT UNDERSTANDING AND
DEVELOPING NEW TREATMENTS FOR
SOME OF THE HORRIBLE MONOGENICS
DISEASES IN PARTICULAR IS SOME
OF THE BEST MONEY WE CAN SPEND.
>> WITH RESPECT TO THE RARE
DISEASES, ONE OF THE THINGS THAT
FASCINATE ME, NOW THAT WE HAVE
THESE GENOMIC TOOLS AND START TO
UNDERSTAND COMMON DISEASE
MECHANISM AND RARE DISEASES MANY
OF THEM TRIGGER COMMON DISEASE
PATHWAYS TO A SPECIFIC ORGAN.
AN EXAMPLE IS GROVER'S DISEASE,
HAILEY-HAILEY DISEASE, DIFFERENT
MECHANISMS THAT STARTED BUT
COMMON DISEASE PATHWAYS, NOW
BEGINNING TO APPLY CLINICAL
TRIALS USING MEB KINASE
INHIBITOR.
RARE DISEASES, AS BOB SAID,
SOMEBODY SAID IN THE PAST, THESE
ARE EXPERIMENTS BY NATURE THAT
GIVE US INSIGHT INTO BIOLOGY,
AND BY "WE," WE ARE THE ONES
THAT CAN WORK ON RARE DISEASES
BECAUSE WE'RE NOT DRIVEN BY
FINANCIAL INTERESTS.
SO I THINK IT'S A GREAT
SCIENTIFIC OPPORTUNITY, A GREAT
OPPORTUNITY TO HELP PEOPLE.
THAT'S WHY I LIKE IT.
>> NO, NO, THAT WAS GREAT.
YOU ANSWERED TWO QUESTIONS.
OTHER MORE COMMENTS ABOUT THIS
TOPIC, ANYTHING ELSE?
YES, TEO?
>> JUST A QUICK FOLLOW-UP.
I REALLY AGREE WITH JINOOS ABOUT
THE PILOTING, THE EFFORT.
IS THERE GOING TO BE A PATIENT
ADVISORY BOARD REVIEW TO PROVIDE
INPUT INTO THE IMPACT OF THE
CLINICAL STUDY?
BECAUSE I THINK THAT WOULD BE
CRITICALLY IMPORTANT IN ADDITION
TO ANY INPUT FROM THIS GROUP.
>> EXCELLENT POINT.
I THINK KATHY IS STILL HERE.
I BELIEVE -- IS THERE A PERSON
WITH LIVED EXPERIENCE ON OUR
AMSC AND AMS -- AS ONE PIECE OF
THAT, BUT THAT COULD ALSO BE A
ROLE FOR -- WE HAVE PEOPLE WITH
LIVED EXPERIENCE INTENTIONALLY
ON COUNCIL, AND THAT COULD BE
ONE OF THE WAYS THAT PERSPECTIVE
IS APPLIED.
EXCELLENT POINT.
LET'S SEE.
I'M NOT SEEING OTHER HANDS UP ON
ZOOM OR IN THE ROOM.
AND I'M JUST DOUBLE CHECKING.
YEAH, WE'RE GOING TO -- PARDON?
BEFORE THE FUN PART?
DARREN HAS A FEW WORDS AND I GET
THE FUN PART.
>> YEAH, I WANTED TO ALSO THANK
EVERYONE WHO CAME TO THE
MEETING, PARTICIPATED IN THIS
OPEN SESSION OF COUNCIL, WHEN
LINDSEY HITS THE GAVEL THAT WILL
CONCLUDE THE OPEN SESSION.
AND WE'LL BEGIN THE CLOSED
SESSION.
REMINDER, YOU SHOULD HAVE
RECEIVED A SEPARATE ZOOM LINK
FOR THE CLOSED SESSION SO WE'LL
CLOSE OUT THIS ZOOM LINK.
THE SESSION OF COUNCIL CLOSED TO
THE PUBLIC IS IN ACCORDANCE WITH
PROVISIONS IN FEDERAL ADVISORY
COMMITTEE ACT FOR REVIEWING
DISCUSSIONS OF GRANTS AND
RELATED INFORMATION, COUNCIL
MEMBERS WILL BE REQUIRED TO
ABSENT THEMSELVES FROM
DISCUSSION DURING ANY REVIEW IF
THEIR PRESENCE CONSTITUTES OR
APPEARANCE TO CONSTITUTE
CONFLICT OF INTEREST.
THAT'S ALL I HAVE TO SAY.
>> I HAVE A PROCEDURAL QUESTION.
GAFFE BEFORE CLOSING LANGUAGE.
>> IT'S SIMULTANEOUS.
I CLOSE THIS MEETING, BANG.
>> I'LL DO THIS FIRST.
ALL RIGHT.
SO, THANK YOU, EVERYONE.
SO THIS CONCLUDES THE OPEN
SESSION OF OUR MEETING.
WE'LL NOW BEGIN THE CLOSED
SESSION, AFTER A BREAK.
AND I WANT TO REMIND EVERYONE
THIS SESSION OF COUNCIL IS
CLOSED TO THE PUBLIC IN
ACCORDANCE WITH PROVISIONS IN
THE FEDERAL ADVISORY COMMITTEE 7174 04:37:34,004 ACT.