EVERYONE JOINING US FOR THE
COMMITTEE ON LYMPHATIC
INFECTIOUS DISEASES.
THIS IS THE SECOND STAKEHOLDER
ENGAGEMENT MEETING WE HAVE AND
WE ARE WELCOMING ALL OUR GUESTS
WHO ARE HERE IN BETHESDA,
MARYLAND AND ZOOM.
THANK YOU FOR YOUR SUPPORT AND
WE ALSO WELCOME EVERYONE
LISTENING THROUGH THE NIH
VIDEOCAST.
I'M SELEN CATANIA IN THE
DIVISION OF CARDIOVASCULAR
SCIENCES.
THE EXECUTIVE SECRETARY OF THE
COMMISSION SERVING AS A LIAISON
BETWEEN THE NCLB AND THE NIH
LEADERSHIP AND REPRESENTING HERE
A LARGE NHLBI SUPPORT STRUCTURE
FOR THE COMMISSION.
I'M GOING TO MAKE A FEW
ADMINISTRATIVE ANNOUNCEMENTS AND
AND HOUSEKEEPING REMINDERS
BEFORE WE START.
FIRST I WANT TO WELCOME OUR
PUBLIC VISITORS FOR THE OPEN
SESSION.
WE'RE LOOKING FORWARD TO YOUR
FEEDBACK.
LISTENERS ON THE VIDEOCAST
ENVIRONMENT CAN USE THE SEND
FEEDBACK AND YOUR QUESTIONS AND
FEED LAB WILL BE RECORDED AND
TAKEN INTO CONSIDERATION AND THE
MEETING IS BEING WEBCASTED AND
REPORTED AND THE VIDEO ARCHIVED
AND AVAILABLE FOR THE PUBLIC AND
OUR NIH COLLEAGUES FOR AN
INDEFINITE AMOUNT OF TIME.
A BRIEF OVERVIEW OF TODAY'S
AGENDA WE HAVE TWO PANEL
SESSIONS A GROUP OF GOVERNMENT
AGENCIES IN THE MORNING AND A
GROUP OF ORGANIZATIONS IN THE
AFTERNOON.
AS THE COMMISSION CONTINUES TO
BETTER UNDERSTAND AND ADDRESS
LYMPHATIC DISEASES, TODAY'S
PANEL ARE ESSENTIAL FOR THEIR
EFFORTS.
WE THANK ALL OUR PANELS.
DURING LUNCH BREAK WE'LL MAKE
NECESSARY SEATING AND VIRTUAL
ARRANGEMENTS FOR THE AFTERNOON
SESSION AND WE WILL REQUEST
PANEL A PARTICIPANTS TO EXIT THE
MEETING FOR THAT PANEL MEETING
COMMENT.
SO WITH THAT, I WANT TO HAND IT
TO THE CO-CHAIRS STEPHANIE
DREYER AND STANLEY ROCKSON WILL
INTRODUCE THE CHARGE AND
PROGRESS OF THE COMMISSION AND
EACH SESSION WILL HAVE TWO PARTS
THE PRODUCTION AND OPEN PANEL
DISCUSSION.
WE REQUEST EVERYONE IN ZOOM TO
HAVE THEIR CAMERA ON AND USE
HAND RAISE FUNCTION DURING
DISCUSSIONS.
ALSO CHAT COMMUNICATIONS WILL BE
SAVED FOR THE RECORD OF THE
COMMISSION.
WITH THAT, IT IS MY GREAT
PLEASURE TO PASS THE MIKE TO
STANLEY ROCKSON ONE OF THE
CO-CHAIRS FOR WELCOMING REMARKS.
>> THANK YOU, SELEN.
>> I'D LIKE TO BEGIN BY
DISCUSSING THE CHARGE OF THE
COMMISSION.
THIS IS A TRANSPARENT AND
INCLUSIVE, ACCESSIBLE PUBLIC
FORUM AND ADDRESS CRITICAL
CHALLENGES IN SCIENTIFIC AND
CLINICAL OPPORTUNITIES INCLUDING
FUNDAMENTAL KNOWLEDGE,
IMPLEMENTATION GAPS AND
DISPARITIES TO UNDERSTAND AND
ADDRESS THE INDIVIDUAL AND
COLLECTIVE BURDENS OF LYMPHATIC
DISEASES.
WE'LL BE GATHERING INPUT FROM A
WIDE AND DIVERSE SPECTRUM OF
STAKEHOLDERS TO IDENTIFY THE
REMAINING GAPS AND CHALLENGES.
WE ARE GOING TO PROVIDE
ACTIONABLE PROGRESS UPDATES AND
THE FINAL REPORT TO THE NHLBI
ADVISORY COUNCIL TO REFLECT
CHALLENGES AND BURDENS AND GAPS
AND OPPORTUNITIES IN LYMPHATIC
SCIENCE AND CLINICAL SCIENCE AND
EDUCATION SHORTFALLS THAT NEED
TO BE REMEDIED.
BY WAY OF INTRODUCTION, OUR
COMMISSION WORKS AS A WHOLE BUT
HAS DIVIDED ITSELF INTO A
VARIETY OF SUBCOMMITTEES AND
MANY OF THE COMMISSIONERS SERVE
ON MULTIPLE OF THESE COMMITTEES.
YOU CAN SEE WE HAVE THOSE
DEVOTED TO SCIENTIFIC
ADVANCEMENT, EDUCATIONAL
IMPERATIVES, MEASURABLE RESULTS,
POTENTIAL TOOLS AND THIRD PARTY
COVERAGE AND COMMUNITY GROWTH
AND SEE THE CHAIR OF EACH
COMMITTEE HIGHLIGHTED.
AND AGAIN MULTIPLE ASSIGNMENTS
AMONG THE COMMISSIONERS.
AT INTRODUCTION WE'LL PROCEED TO
PANEL A ADDRESSING THE GAPS AND
BARRIERS IN THE FIELD OF
LYMPHATIC DISEASES AND LYMPHATIC
RESEARCH.
I'M GOING TO BEGIN BY
INTRODUCING OUR FIRST
STAKEHOLDER, DR. CECELIA
DUPECHER MEDICAL RESEARCH
PROGRAM, CONGRESSIONALLY
DIRECTED U.S. ARMY MEDICAL
RESEARCH AND DEVELOPMENT
COMMAND, DEPARTMENT OF DEFENSE
TO MAKE INTRODUCTORY COMMENTS.
S
>> IT'S ALL YOURS.
>> CAN EVERYONE HEAR ME?
IT'S A PLEASURE TO BE HERE THIS
MORNING.
I'M EXCITED TO TALK ABOUT OUR
ORGANIZATION AND TO BE PART OF
THIS PANEL.
I HAVEN'T GOTTEN AN ANSWER ON
THE CHAT.
ARE YOU GOING TO BE SHARING THE
SLIDES OR ARE THE PRESENTERS
GOING TO BE PRESENTING THE
SLIDES?
>> WE'RE SHARING THE SLIDES FOR
YOU.
ARE YOU SEEING THE ZOOM
>> PERFECT.
SHALL I JUST HOP IN?
I'M CECELIA DUPECHER ONE OF 35
ORGANIZATIONS CALLED
CONGRESSIONALLY DIRECTED MEDICAL
RESEARCH PROGRAMS.
I'LL GIVE AN OVERVIEW OF THE
ORGANIZATION AND GO OVER THE
PROGRAM CYCLE AND REVIEW PROCESS
AND THEN HOP IN THE PROGRAM THAT
HAS LYMPHATIC DISEASE AND
LYMPHEDEMA AS A DIRECTED TOPIC
AREAS FOR RESEARCH.
THERE'S A LOT OF INFORMATION ON
THE SLIDES.
IN THE INTEREST OF TIME I HEARD
WE'RE RUNNING LATE SO I'LL TRY
TO BE AS BRIEF AS I CAN.
IF YOU HAVE QUESTIONS FEEL FREE
TO REACH OUT AND I'M SURE WE CAN
DISSEMINATE THE SLIDES AFTER
THIS BRIEF.
WE STARTED IN 1992 WITH
GRASSROOTS ADVOCACY AND THE
APPROPRIATION WAS MANAGED BY THE
U.S. ARMY AT THE TIME AND HOW
THE FIRST PROGRAM OF THE
ORGANIZATION STOOD UP THE BREAST
CANCER RESEARCH PROGRAM IN 1992.
AND WE HAVE STEADILY GROWN SINCE
THEN WITH FURTHER APPROPRIATIONS
FROM CONGRESS.
WE FUNDED IMPACTFUL RESEARCH WE
LIKE TO SAY RISKY RESEARCH THAT
OTHER ORGANIZATIONS MAY NOT
FUND.
WE CAN FUND ANYWHERE IN THE
WORLD SO WE'RE NOT JUST LIMITED
TO THE UNITED STATES.
AND AWAY USE OUR TIER 2 REVIEW
PROPOSAL.
THIS IS THE HALLMARKS OF THE
ORGANIZATION IN BROAD STROKES I
WANT TO SHOW UNDER THE HEADER OF
IMPACT.
WE DO NOT REQUEST THE FUNDS.
THEY'RE ADDED THROUGH THE
DEFENSE APPROPRIATION BILL.
WE PUBLICALLY ANNOUNCE AND
COMPETE OUR FUNDING
OPPORTUNITIES AND FOLLOW THIS
NATIONAL ACADEMY OF MEDICINE
RECOMMENDS TO TIER 2 REVIEW
PROFESSION WE'LL DISCUSS LATER.
WE STAND ON THE FOUNDATIONS OF
GRASSROOTS ADVOCACY SO THE
CONSUMERS WHICH ARE PATIENTS
LIVING ONE THE DISEASE ARE THE
TRUE NORTH OF THE ORGANIZATION
AND WILL HAVE AN EQUAL VOTE AS
SCIENTIST REVIEWERS DURING OUR
PREREVIEW AND PROGRAMATIC
REVIEW.
AND IN TERMS OF ADMINISTRATIVE
HOW WE OBLIGATE FUNDS WE RECEIVE
OUR APPROPRIATIONS ANNUALLY
THERE ARE WE OBLIGATE OUR FUNDS
UP FRONT.
WE DON'T HAVE LIMITED OUT YEAR
BUDGET COMMITMENTS.
THESE ARE THE 35 PROGRAMS YOU
SEE LISTED FOR ORGANIZATION.
FOR FY24.
ON THE RIGHT YOU'LL SEE THE
RESPECTIVE APPROPRIATION FOR
EACH ONE OF THE PROGRAMS.
YOU CAN SEE WE HAVE PROGRAMS
MUCH SMALLER IN SIZE IN TERMS OF
MILLIONS AND LARGER PROGRAMS.
THERE'S LISTED IN ALPHABETICAL
ORDER WE HAVE RARE CANCERS AND
TRAUMATIC VISION AND THERE'S A
WIDE RANGE OF PROGRAMS HERE.
TODAY I'LL TALK ABOUT THE PEER
REVIEWED MEDICAL RESEARCH
PROGRAM.
IT'S HERE ON THE RIGHT SIDE.
YOU CAN SEE IT HAS 42 TOPIC
AREAS.
I'LL TELL YOU A LITTLE BIT HOW
THE TOPIC AREAS WORK.
WE HAVE BEEN AROUND FOR QUITE A
WHILE AND OUR ANNUAL
APPROPRIATION IS $370 MILLION.
SO NOW GOING TO OUR PROGRAM
CYCLE AND REVIEW PROCESS.
LIKE I SAID, WE'RE APPROPRIATED
YEARLY SO OUR FUNDING
OPPORTUNITIES CAN ONLY BE
RELEASED AFTER THE APPROPRIATION
BILL IS SIGNED INTO LAW.
SO WHEN WE ARE IN CONTINUING
RESOLUTION WHICH IS NOW IT'S
ABOUT TO HAPPEN IT DOES COMPRESS
OUR TIME LINES.
WE GO THROUGH DEPENDING ON THE
AWARD MECHANISM EACH PROGRAM IS
A STAND ALONE PROGRAM.
WE CAN EACH INDIVIDUALLY RELEASE
OUR FUNDING OPPORTUNITY AND NOT
ONE TO THE OVER ALL ORGANIZATION
IT'S FOR EACH OF THE INDIVIDUAL
PROGRAMS.
WE'LL GO THROUGH A
PRE-APPLICATION SCREENING
PROCESS AND PEER-REVIEW MANAGED
BY A CONTRACT WITH GENERAL
DYNAMICS AND GO TO PROGRAMATIC
REVIEW.
WE DON'T HAVE A CENTER FOR
SCIENTIFIC REVIEW.
OUR STANDING PANEL ARE BASED ON
THE PROPOSALS WE RECEIVE.
ONE OF THE THINGS I'D LIKE TO
CALL YOUR ATTENTION HERE YOU'LL
SEE ON THE LEFT PROGRAMMATIC
PANEL IN BROWN.
THIS IS A BOARD OF ADVISORS PART
OF EACH ONE OF THE PROGRAMS.
THEY'LL HAVE THAT AND IT IS
REPRESENTED BY GOVERNMENT
EMPLOYEES, OTHER STAKEHOLDERS,
CONSUMERS AND A VARIETY OF
EXPERTISE NEEDED.
AND THEY WILL HAVE INPUT
THROUGHOUT THE ENTIRETY OF THE
PROGRAM CYCLE.
AFTER WE SELECT PROPOSALS.
THERE'S GOING TO BE A
RECOMMENDED FUNDING LIST AND THE
DECISION AUTHORITY AND THEN
WE'LL ENTER TO THE NEGOTIATIONS.
SO THIS IS THE TWO-TIER REVIEW
PROCESS.
LIKE I SAID WE HAVE PEER-REVIEW
AND GO ONE STEP FURTHER WITH THE
PROGRAMMATIC REVIEW.
SIMILAR TO WITH COUNCIL AT THE
NIH BUT DO NOT HAVE A FUNDING
LINE.
WE START WITH THE PEER REVIEW
AND THE MERIT OF PROPOSAL AND
THE SUMMARY STATEMENT, A
DOCUMENTATION OF STRENGTHS AND
WEAKNESSES AND THEIR PROGRAMATIC
PANEL CAN LOOK INTO HOW WELL IT
ADDRESSES THE INTENT OF THE
AWARD MECHANISM AND POTENTIAL
FOR IMPACT AND OF THOSE
SELECTED.
WE'LL COME OUT WITH A
RECOMMENDED FOR FUNDING LIST.
SO WE DO NOT HAVE A FUNDING
LINE.
LIKE I SAID THE SIDE SUMMARIZES
THE CONSUMER INVOLVE MANY.
THEY'RE INVOLVED IN EVERY STEP
OF THE WAY AND WE HAVE CONSUMERS
THAT ARE FOR EVERY PROGRAMATIC
PANEL AND PEER REVIEW PANEL
WE'LL HAVE CONSUMERS BASICALLY
LOOKING AT THE IMPACT THAT EACH
PROPOSAL WILL HAVE ON THEIR
COMMUNITY.
THEIR VOTE IS AN EQUAL VOTE AS A
SCIENTIST REVIEWER.
NOW WE HAVE 35 PROGRAMS.
WE'LL FOCUS ON THE PEER REVIEWED
MEDICAL RESEARCH PROGRAM.
THIS IS WHAT I MANAGE.
THE LARGEST PROGRAM IN THE
ORGANIZATION AND THAT HAS TOPIC
AREAS RELATED TO WHAT WE'RE
DISCUSSING HERE TODAY.
SO THIS PROGRAM STARTED IN 1999
WITH A CHARGE TO ADDRESS
DISEASES AND CONDITIONS WITH
RELEVANCE TO MILITARY HEALTH.
PART OF OUR CONGRESSIONAL
LANGUAGE FOR US TO SUPPORT
RESEARCH OF CLEAR SCIENTIFIC
MERIT AND THAT'S EMBEDDED IN THE
CONGRESSIONAL LANGUAGE EACH YEAR
AND RELEVANCE TO MILITARY HEALTH
IN SPECIFIED TOPIC AREAS.
UNLIKE SOME PROGRAMS YOU SAW IN
THE LIST THAT WOULD HAVE A
PRESCRIPTIVE LANGUAGE AND
DIRECTIVE, CDMRP OPERATES IN THE
PREMISE OF THESE TOPIC AREAS.
WE RECEIVE FUNDING APPROPRIATION
AND THE TOPIC AREAS END UP IN
THE PROGRAM.
IT'S A REFLECTION OF LOBBYING
EFFORTS, GRASSROOTS,
PROFESSIONAL LOBBYISTS BUT
ULTIMATELY CONGRESS'S DECISION.
WE HAVE LONG STANDING TOPIC
AREAS IN THE PROGRAM AND HAVE
TOPIC AREAS THAT VARY EACH YEAR.
I'M LISTING THOSE FOR FY24 AND
IN BLUE HAVE NEVER BEEN
ASSIGNED.
THE MAJORITY OF THE TOPIC AREAS
HAVE BEEN LONG STANDING WITH THE
PROGRAM.
IN FY24 WE HAD LYMPHEDEMA AND
LYMPHATIC DISEASES.
THIS IS THE ONE I'M GOING TO
SHOW THE PROPOSALS WE ENDED UP
FUNDING.
LYMPHATIC DISEASES ENDED FALLING
OFF CONGRESSIONAL LANGUAGE AND
STAYED ONLY WITH LYMPHEDEMA.
AND WE WILL ALLOCATE THEM IN THE
PORTFOLIOS YOU SEE THEY ALIGN
WITH THE MAJOR AREAS OF
SCIENTIFIC RESEARCH.
HOW WE DO THAT WE DISCUSS WITH
THE PROGRAMATIC PANEL AND THE
PORTFOLIO DRIVEN APPROACH ALLOWS
US TO TAKE A MORE TARGETED
APPROACH TO FUNDING.
WE CAN LOOK AT THE GAPS IN EACH
ONE OF THESE AND PROVIDES A
LITTLE BIT MORE CONTINUITY FOR
TOPIC AREAS THAT FALL OFF MAKING
SURE WE HAVE BROAD GOALS FOR
EACH.
LIMB FA -- LYMPHEDEMA AND
LYMPHATIC DISEASES WERE IN THE
PORTFOLIO.
WE DEVISE THESE WITH OUR
PROGRAMATIC PANEL AND REACH OUT
TO ADVOCACY GROUPS TO CAPTURE
THE CONSENSUS BETWEEN THE TOPIC
AREAS ALIGNED IN THE PORTFOLIO
AND SOLICITING FOR THE RESEARCH
THAT'S NEEDED.
THE GOALS WILL STAN ACROSS THE
CONTINUUM OF CARE FOUNDATIONAL
PREVENTION, DIAGNOSIS, TREATMENT
AND AN INVESTIGATOR BY LAW THEY
HAVE TO ADDRESS TOPIC AREAS
ASSIGNED TO THE PROGRAM AND MAKE
THEM ADDRESS ONE OF THE
STRATEGIC GOALS ASSIGNED TO THAT
PORTFOLIO.
THIS IS JUST AN EXAMPLE OF WHAT
THIS WOULD LOOK LIKE IN OUR
FUNDING OPPORTUNITIES.
EACH PROGRAM RELEASES THEIR OWN
FUNDING OPPORTUNITY.
EACH OF OUR FUNDING
OPPORTUNITIES IS GOING TO LIST
TOPIC AREAS ELIGIBLE.
THE PORTFOLIO YOU'LL SEE AN
EXAMPLE OF THE TOPIC AREAS
ASSIGNED TO INTERNAL MEDICINE IN
FY24 AND THEN EXAMPLES OF THE
STRATEGIC GOALS AND WHAT WE'RE
LOOKING FOR.
THE GOALS ARE BROAD AND MEANT TO
BE BROAD AND WE'RE LOOKING AT
CONSENSUS OF THE TOPIC AREAS AND
TO MAKE SURE WE'RE NOTE LIMITING
CREATI
CREATIVITY.
WE HAVE THE ABILITY TO SOLICIT A
BROAD RANGE OF RESEARCH IN
DIFFERENT AREAS OF PRODUCT
DEVELOPMENT.
WE'RE MAKING SURE THAT WE KEEP
THEM BROAD AND THAT EVERY NEW
PROPOSAL HAS A VENUE TO COME IN
AND GET FUNDING THROUGH THE
PROGRAM.
HERE ARE THE OTHER EXAMPLES OF
STRATEGIC GOALS THAT WE HAVE
UNDER TREATMENT IN EPIDEMIOLOGY.
SO LIKE I SAID, WE CAN ALL EACH
INDIVIDUALLY RELEASE THE FUNDING
OPPORTUNITIES FOR THIS PROGRAM.
WE RELEASED SIX FUNDING
OPPORTUNITIES AND BASIC RESEARCH
ALL THE WAY TO CLINICAL TRIALS.
WE HAVE TWO AWARDS IN BASIC
RESEARCH THE DISCOVERY AND
INVESTIGATOR RESEARCH AWARD THE
IIRA IS SIMILAR TO THE RO1 TO
THE NIH.
THEN WE MOVE TO TRANSLATIONAL
RESEARCH.
WE HAVE THE IMPACT AWARD THAT'S
BASICALLY LOOKING AT USE
INSPIRED SCIENCE MORE DEVELOPED
AND TTGA DEVELOPMENT AWARD AND
THAT IS BASED ON THE PREMISE BY
THE END OF THE PERIOD OF
PERFORMANCE THE P.I. WOULD BE
READY TO SUBMIT AN IND.
THEN WE MOVE TO OUR CLINICAL
TRIALS.
ONE ON CLINICAL RESEARCH AND
EVERYTHING NOT FDA REGULATED AND
OUR CLINICAL TRIAL AWARD WHICH
IS POPULAR.
WE DO NOT HAVE A DIRECT COST
LIMIT AND IT'S REALLY A LOT OF
WHAT THE PMRP FUNDS.
SO THESE ARE THE AWARDS FUNDED
SO FAR FOR FY23.
THE YEAR WE HAVE LYMPHEDEMA AND
LYMPHATIC DISEASES.
FOR FY24 THE PROPOSALS ARE STILL
BE REVIEWED.
WE HAVE A RECOMMENDED FOR
FUNDING LIST LATER IN THE YEAR
FOR SOME AWARD MECHANISMS AND
NEXT YEAR FOR THE CLINICAL
TRIALS.
YOU CAN SEE SO FAR, WE HAVE
FUNDED A LITTLE BIT SHY OF $7
MILLION.
WE HAVE THREE DISCOVERY AWARDS
WHICH ARE ONES WITH DIRECT COST
AND INNOVATIVE RESEARCH FOR 250K
AND A BASIC AN IIRA FOR $2.6
MILLION AND A CLINICAL TRIAL.
I'M HAPPY TO PROVIDE MORE
INFORMATION IF ANYBODY FEELS
COMPELLED TO REACH OUT TO ME.
I'LL PAUSE HERE.
I KNOW IT WAS A LOT OF
INFORMATION AND HOPE IT WAS
HELPFUL AND GRATEFUL TO BE HERE
TO TALK ABOUT CDMRP.
>> THANK YOU FOR THE
PRESENTATION.
WE'LL HAVE QUESTIONS AND ANSWERS
DURING THE PERIOD TO FOLLOW.
NEXT I'LL INTRODUCE DR. AMY
JENKINS AND DR. KIM STEELE
REPRESENTING ARPA-H.
>> CAN EVERYBODY HEAR ME OKAY?
WONDERFUL.
THANK YOU.
THIS MORNING KIMBERLY AND I WILL
DO A TAG TEAM.
I'LL GIVE EVERYBODY AN OVERVIEW
OF ARPA-H, OUR MODEL.
SIMILAR TO WHAT YOU HEARD FROM
CEASE CELIA AND KIMBERLY WILL
TALK ABOUT OUR TWO PROGRAMS IN
LYMPHATIC DISEASE.
ARPA-H IS A FAIRLY NEW
ORGANIZATION WITHIN THE
DEPARTMENT OF HEALTH AND HUMAN
SERVICES.
WE HAVE A SINGULAR MISSION TO
ACCELERATE BETTER HEALTH
OUTCOMES FOR EVERYONE.
SO ARPA-H IS STOOD UP IN THE
DIVISION OF THE DARPA THE
DEFENSE ADVANCE RESEARCH AGENCY
AN AGENCY I WAS AT 10 YEARS IN
THE BIOLOGICAL TECHNOLOGIES
OFFICE PRIOR TO ARPA-H.
DARPA TO GIVE PRIMER ON THIS WAS
STOOD UP IN 1958 IN RESPONSE TO
THE LAUNCH OF THE SPUTNIK
SATELLITE.
THE ORGANIZATION WAS STOOD UP TO
FUND RESEARCH THAT WAS
HIGH-RISK, HIGH-REWARD,
HIGH-IMPACT AND MEANT TO BE
FOCUSSED RESEARCH TO MAKE
INVESTMENTS IN TECHNOLOGIES FOR
NATIONAL SECURITY.
THAT'S WHAT DARPA HAS BEEN DOING
ABOUT 65 YEARS.
IN THE SPRING OF 2022, PRESIDENT
BIDEN SAID THE ADMINISTRATION
WOULD START AN ORGANIZATION THAT
WOULD TAKE THAT MODEL OF PIVOTAL
FOCUSSED INVESTMENTS AND DARPA
HAS APPLIED IT TO THE HEALTH
CARE CENTER.
THE STAND UP OF ARPA-H BEGAN IN
SPRING OF 2022 IN THE SAME
MODEL.
WE'LL USING THE SLIDE TO TALK
ABOUT IT SOMETIMES THE GOAL IS
TO MAKE ACCELERATED FOCUSSED
INVESTMENTS.
MOST THE TIME THOSE FAIL AND
WE'RE HITTING THE ACCELERATOR
AND YOU'LL SEE THE TIME LINES IN
KIMBERLY'S SLIDES.
OFTEN THINGS WE'RE DOING ARE NOT
ACHIEVABLE BUT OCCASIONALLY YOU
ARE SUCCESSFUL AND DISCOVER
THINGS LIKE THE INTERNET,
STEALTH TECHNOLOGY AND
SELF-DRIVING CARS AND mRNA
VACCINES AND THE MODEL WORKS TO
MAKE THESE LEAPS IN TECHNOLOGY.
THAT'S WHAT WE'RE HERE FOR.
I'LL TALK A LITTLE BIT ABOUT
WHAT THAT MODEL IS WHEN I SAY
WE'RE STOOD UP IN THE DIVISION
OF DARPA, WHAT IS IT AND WHAT'S
DIFFERENT ABOUT ARPA-H?
WE'RE A FUNDING AGENCY.
WE HAVE NO INTERNAL RESEARCH
LABS AND OUR PROGRAM MANAGERS
BRING IDEAS TO THE AGENCY.
WE'RE NOT IN THE HEALTH SCIENCE
FUTURES OFFICE AT ARPA-H.
I'M NOT DRIVING THE MISSION OR
SAYING WHAT WE WILL OR WILL NOT
WORK ON.
OUR PROGRAM MANAGERS COME IN AND
SAY THIS A PROBLEM WE NEED TO
ADDRESS AND IF I AGREE THEY'RE
THE RIGHT PERSON TO ADDRESS THE
PROBLEM THEY COME UP AND DRIVE
THAT PROGRAM.
SO IT'S IT'S A BOTTOM-UP AGENCY
AND NOT LOOKING FOR THE NEXT
INCREMENTAL STEP BUT TAKE THE
LEAP AND WILL PROBABLY MISS,
MANY TIMES WE'LL MISS BUT IN THE
PROCESS OF TAKING THAT LEAP WE
LEARN A LOT ALONG THE WAY AND
SOMETIMES WE'RE SUCCESSFUL.
THAT'S PART OF THE GOAL HERE.
WHAT SETS US APART IS OUR
PROGRAM MANAGERS ARE WHO DRIVE
THE SCIENCE BEHIND THE AGENCY.
THEY HAVE FULL AUTONOMY WITHIN
THEIR PROGRAM.
WE DON'T DO STUDY SECTIONS, WE
DO HAVE GOVERNMENT REVIEW OF OUR
PROPOSALS, THREE REVIEWERS.
ULTIMATELY THE DECISION LIES
WITH KIMBERLY AND ME AS HER
CHECK TO NOT DO ANYTHING TOO
CRAZY BUT WE WANT TO FUND THE
RISKY IDEAS AND WE ARE TRUSTING
HER TO FIND AND FUND THOSE RISKY
IDEAS.
OUR PROGRAM MANAGERS AND OUR
TECHNICAL LEADERSHIP HAVE TERM
LIMITS.
EVERYBODY IN THE ORGANIZATION
HAS TO LEAD.
WE HAVE THREE-YEAR TERMS AND CAN
BE RENEWED FOR ADDITIONAL THREE
YEAR FOR MAXIMUM OF SIX YEARS
AND THEN ALL LEAVE.
THAT'S SET UP ON PURPOSE SO THE
IDEA IS CONSTANTLY TRYING TO
TAKE THE NEXT LEAP.
WE'RE VERY INTERESTED IN
LYMPHATICS BECAUSE KIMBERLY SAID
WE SHOULD BE AND WE BELIEVED HER
BUT SIX YEARS FROM NOW WE WON'T
BE IN THE LYMPHATIC SPACE.
WE'LL PUT THE MONEY WE'LL PUT IN
AND THE HOPE IS TO MAKE RADICAL
CHANGE IN THE FIELD AND PUSH THE
FIELD FORWARD BY DECADE TO CATCH
TO WHERE THE FIELD IS FOR OTHER
DISEASES LIKE CARDIOVASCULAR
DISEASE BUT WON'T FUND THIS IN
PERPETUITY AND THE EFFORTS WILL
END AND WILL NOT BE RENEWING THE
EFFORTS.
I'LL BRIEFLY TALK ABOUT WHAT ARE
STAKEHOLDERS AND THE HEALTH ECO
SYSTEM IN WE WORK WITH OUR
PATIENT GROUPS AND HEALTH CARE
PROVIDERS TO HEAR WHAT THE
PROBLEM IS.
DOWN ON THE LEFT SIDE IS WHAT I
WANT TO TALK ABOUT THE MOST OUR
PERFORMERS.
WE DON'T CALL THE TEAMS WE FUND
GRANTEES BECAUSE WE DON'T ISSUE
GRANTS.
WE ONLY ISSUE OTHER TRANSACTIONS
OR COOPERATIVE AGREEMENTS BUT
WORK WITH ACADEMIA AND INDUSTRY
SMALL AND LARGE AND NONPROFITS
TO REACH THE GOALS OF THE
ORGANIZATION.
THESE ARE OUR INITIAL FOCUS
AREAS ALIGN TO OUR FOUR MISSION
OFFICES, HEALTH SCIENCE FEATURES
IS THE OFFICE I'M THE DIRECTOR
OF KIMBERLY IS A PROGRAM MANAGER
IN.
I WON'T GO IN OTHER OFFICES IN
THE INTEREST OF TIME YOU CAN
GUESS A PROACTIVE AND
PREVENTATIVE AND LOTS OF
MANUFACTURING AND SYSTEMS
TECHNOLOGIES IN OTHER OFFICES.
I HAVE MENTIONED IDEATION AT
ARPA-H STARTS AND ENDS WITH THE
PROGRAM MANAGER.
THEY COME IN WITH THE IDEA AND
THE PROGRAMS ARE FAIRLY LARGE
INITIATIVES.
YOU'LL HEAR TWO OF KIMBERLY'S
TODAY AND WHERE WE DO THE BULK
OF OUR FUNDING.
WE'LL DO A SOLICITATION AND
TEAMS WILL COME AND SUBMIT THEIR
PROPOSALS TO THAT PROGRAM AND TO
THE REVIEW PROCESS AND KIMBERLY
WILL CHOOSE THE TEAMS SHE WANTS
TO FUND IN THAT PROGRAM AREA.
WE FUND IDEAS THAT COME
OCCASIONALLY.
WE DO HAVE AN OPEN CALL FOR
PROPOSALS ON THE WEBSITE WE CALL
INNOVATIVE SOLUTIONS OFFERINGS.
IT'S ESSENTIALLY AN OPEN CALL
RFP.
PEOPLE CAN BRING OUTSIDE IDEAS.
WE DON'T WANT TO MISS THEM
BECAUSE PROGRAM MANAGE ERS MAY T
BE IN THAT SPACE BUT ISSUED
THROUGH OUR PROGRAMS.
I TALKED BRIEFLY ABOUT SOME OF
OUR PROGRAMS.
THIS IS AN EXCITING SLIDE FOR US
BEING WE STOOD UP IN 2022 OUR
DIRECTOR JOINED THE AGENCY IN
THE FALL OF 2022 AND WE OPENED
YOU'RE FIRST ANNOUNCEMENT FOR
FUNDING IN THE SPRING OF 2023
WHEN I JOINED AND OUR FIRST
PROGRAM WAS LAUNCHED IN APRIL OF
'23 CALLED THE NYTRO PROGRAM
AROUND OSTEOARTHRITIS AND SEE
THE VARIETY OF PROGRAMS
LAUNCHED.
I'LL QUICKLY PULL OUT A COUPLE
THINGS THAT ARE INTERESTING IN
THE GRAPHS I CAN DISCUSS.
WE HAVE THE OSTEOARTHRITIS
PROGRAM CALLED NYTRO AND THE EYE
TRANSPLANTATION AND RETINAL
REGENERATION PROGRAM.
WE HAVE EFFORTS AROUND BIO
PRINTING OF ORGANS AND YOU'LL
HEAR EXCITING IDEAS IN THE
LYMPHATIC SPACE FROM KIMBERLY.
A LITTLE BIT OF A SMATTERING.
WE'RE FUNDING HOPEFULLY CRAZY
OUT THERE IDEAS THAT PEOPLE SAY
THAT'S CRAZY TRANSPLANTING AN
EYE AND TRYING TO GET THE
RETINAL NERVE TO REGENERATE AND
WE UNDERSTAND WE MAY NOT BE
SUCCESSFUL.
BUT THAT'S THE GOALS OF OUR
AGENCY TO PUT THOSE SHOTS ON
GOALS AND SEE WHAT ADVANCES WE
CAN MAKE AND IF SUCCESSFUL PUSH
THE FIELD FORWARD BY SEVERAL
DECADES.
I'LL STOP HERE.
THAT'S OUR MODEL VERY FOCUSSED
INVESTMENTS AND I'LL LET
KIMBERLY TALK YOU THROUGH A
COUPLE OF HER PROGRAMS TO MAKE
IT MORE REALISTIC WHAT THE
PROGRAMS LOOK LIKE.
OVER TO YOU.
>> HI, EVERYONE.
IT'S WONDERFUL TO BE HERE.
AND I'M GRATEFUL TO BE HERE AND
GRATEFUL TO DR. JENKINS FOR
BEING A REPRESENTATIVE OF ARPA-H
AS PROGRAM MANAGER.
FOR THOSE WHO MAY NOT KNOW ME
BEFORE I JUMP IN THE PROGRAMS,
AND A LOT OF YOU HERE DO KNOW ME
BUT FOR THOSE WHO DON'T, I AM A
SURGEON BY TRADE.
I WAS AT JOHNS HOPKINS FOR 14
YEARS AND MY AREA OF EXPERTISE
IS IN BARIATRIC SURGERY AND TALK
TRAUMA AND CRITICAL CARE CALLS
AT HOPKINS AND I RAN A
TRANSLATIONAL LAB IN GUT-BRAIN
AXIS AND OBESITY MEDICINE SO AN
ACADEMIC PERSON.
IN 2016 OUR LIFE CHANGED.
MY FAMILY'S LIFE.
I HAVE TWO BOYS AND THE OLDEST
WAS DIAGNOSED AFTER A YEAR-LONG
DIAGNOSTIC ODYSSEY WITH AN ULTRA
RARE LYMPHATIC DISEASE AND WE
WERE TOLD NOT TO READ ABOUT IT
AND GO HOME BECAUSE IT WAS SO
DAUNTING.
LUCKILY I WAS IN A PLACE WHERE I
COULD GET THE HELP I NEEDED.
I REACHED OUT TO EVERYONE AND
ANYONE.
SOME OF YOU SITTING AT THE TABLE
AND YOU RESPONDED QUICKLY.
WHAT I LEARNED THAT NAVIGATING
THE HEALTH CARE SYSTEM WAS TOUGH
FOR A MOM AND PHYSICIAN.
WHAT WAS IT LIKE FOR OTHER
PARENTS?
IT WAS A SYSTEM WE DON'T LEARN
ENOUGH ABOUT OR APPRECIATE
ENOUGH IN MEDICAL SCHOOL AND
SURGERY.
I DIDN'T LEARN ABOUT THE
LYMPHATIC SYSTEM.
I KNEW TO STAY AWAY FROM IT.
IN 2019 I STEPPED AWAY FROM MY
POSITION AND WORKED WITH
NONPROFITS.
BECAUSE I WANTED TO HELP
RESEARCHERS.
I SPENT ABOUT A YEAR IN A SMALL
NONPROFIT AND WAS FRUSTRATED
BECAUSE THEY COULD ONLY DO SO
MUCH AND THE SCIENCE WAS NOT
MOVING FAST ENOUGH WHERE WE
NEEDED TO BE FOR MY CHILD AND
THE COMMUNITY.
SO I TRIED MY OWN NONPROFIT AND
THAT DIDN'T WORK BECAUSE I AM
NOT A FUNDRAISER.
I TRIED.
WE DID GET SOME YOUNG PEOPLE
INTO THE LAB AND WE NOW HAVE A
MOUSE MODEL AND HAVE A BIOMARKER
THAT WE WOULDN'T HAD WE NOT
WORKED ON IT BUT IT WASN'T
ENOUGH.
THEN I WORKED WITH LYMPHATIC
EDUCATIONAL RESEARCH NETWORK AND
I WORKED ON THE REGISTRY AND I
WAS SPECIAL PROJECTS DIRECTOR
FOR A WHILE AND I HEARD ABOUT
ARPA-H.
IT TOOK ME TWO YEARS TO GET
THROUGH THAT TO UNDERSTAND IT
BUT I'M HERE NOW BECAUSE ARPA-H
LISTENED AND I'M A PROGRAM
MANAGER WITH TWO PROGRAMS.
THE FIRST IS LIGHT.
WE LAUNCHED THIS ONE AND WE ARE
NOW IN A SOURCE SELECTION
PROCESS EXPECTING OUR FULL
PROPOSALS AND THAT'S LYMPHATIC
IMAGING AND GENOTYPING AND
PHENOTYPING TECHNOLOGY.
HAVING MET WITH ALL KINDS OF
STAKEHOLDERS ACROSS THE NATION
AND INTERNATIONALLY WE CAME UP
WITH THREE REAL CHALLENGES,
BIOMARKERS, IMAGING, BEING
HIGHLY INVASIVE, NOT VERY
ACCESSIBLE AND HIGHLY
SPECIALIZED.
AND GENETICS.
WE'RE YEARS BEHIND.
SO WE'VE CREATED THREE FOCUS
AREAS OR TECHNICAL AREAS.
THE FIRST ONE IS DIAGNOSIS AND
MONITORING THROUGH BIOMARKER
DISCOVERY.
THE SECOND IS IMAGING
TECHNOLOGIES AND THE THIRD IS
PREVENTION, PREDICTION AND
DIAGNOSTIC CONFIRMATION THROUGH
GENETICS AND EPIGENETICS AND
MODELS OF LYMPHATIC DYSFUNCTION.
NOW, AS I SAID WE'RE JUST IN THE
MIDDLE OF OUR FULL PROPOSALS
COMING IN.
WE'RE REALLY LOOKING FORWARD TO
SEE WHERE THAT TAKES US.
THEN TOMORROW WE WILL BE HOLDING
A PROPOSAL WHILE HEADING ON A
PLANE TO NASHVILLE, TENNESSEE.
OUR SECOND PROGRAM WAS LAUNCHED
CALLED GROUNDBREAKING LYMPHATIC
INTERVENTIONS AND DRUG
EXPLORATION FOCUSSED ON
THERAPEUTICS AND LAUNCHED
TOGETHER A FEAT IN ITSELF AND
CONVINCED LEADERSHIP IT WAS
IMPORTANT TO HAVE TWO PROGRAMS
DIAGNOSTICS AND THERAPEUTICS
RUNNING IN PARALLEL SO OUR
RESEARCHERS COULD INFORM EACH
OTHER WITH COLLABORATION.
IN THIS PROGRAM WE'RE GOING TO
WORK ON TWO TECHNICAL AREAS.
THE FIRST ONE IS PHYSICAL
INTERVENTIONS.
YOU, SURGERY, WEARABLES.
WHAT WAYS CAN WE IMPROVE THE
LYMPHATIC SYSTEM THROUGH
PHYSICAL INTERVENTION AND THE
SECOND IS NORMALIZING FUNCTION
THROUGH PHARMACOLOGIC GENE AND
CELL THERAPIES.
WE ARE DOING OUR PROPOSALS TODAY
AND TOMORROW AND WE'LL BE
SELECTING OUR SOLUTION SUMMARIES
SOON AFTER THAT.
OKAY THIS IS WHAT I MEANT ABOUT
COLLABORATION.
WE'RE ASKING WHAT IF WE CAN MAKE
THE INVISIBLE, VISIBLE?
WHAT'S IT MEAN?
WE WANT TO SHINE A LIGHT ON THE
LYMPHATIC SYSTEM.
IN GLIDE WE'RE TARGETING THE
LYMPHATIC SYSTEM THROUGH FIZZ
Y
-- PHYSICAL INTERVENTIONS AND
HOPING TO RUN THE PROGRAMS IN
PARALLEL.
BEFORE I GO ON TO THAT, EXCITED
TO IT BE HERE BECAUSE AS
DR. JENKINS MENTIONED, I EXPIRE
IN THREE YEARS, HOPEFULLY SIX.
THE PROGRAMS WILL END AND WE'LL
NEED TO DERISK RESEARCH THAT'S
REALLY IMPORTANT BUT HASN'T BEEN
FUNDED YET AND TRANSITION IT
OVER.
HOW DO WE TRANSITION IT OVER?
BY PARTNERS AND COLLABORATIVE
EFFORTS AND NOT REINVENTING THE
WHEEL.
FINALLY JUST AS SMALLER PROGRAMS
THAT WE'VE ALREADY STARTED TO
FUND OR ARE IN THE PROCESS OF
WORKING ON FUNDING, ONE WAS THE
SPRINT FOR WOMEN'S HEALTH AND MY
TEAM HAD THE OPPORTUNITY TO
REPRESENT TOPIC NUMBER 4, WHICH
WAS ADVANCING WOMEN'S BRAIN
HEALTH THROUGH TARGETING THE
LYMPHATIC SYSTEM AND DETERMINING
WHO WILL BE AWARDED BUT I CAN
LET YOU KNOW SOME TOPICS THAT
CAME IN AND THE PROJECTS ARE
FABULOUS AND WE'RE VERY EXCITED.
WE HAVE DIAGNOSTICS AND
THERAPEUTICS THAT WE'LL BE
WORKING ON.
WE ALSO HAVE SMALLER BUSINESS
OPPORTUNITIES AND I REMEMBER
LAST TIME I JUMPED IN AND STAN
WAS ABLE TO HELP OUT.
WE HAVE AN AMBULATORY EDEMA
MONITOR SOLICITED FOR AND IN THE
MIDDLE OF SOURCE SELECTION.
WE HAD GREAT TOPICS AND
HOPEFULLY ANNOUNCING THOSE SOON.
I CAN ANNOUNCE THIS ONE.
WE HAVE TWO AWARDEES FOR OUR
SMALL BUSINESS TOPIC CALLED
LYMPHOLAB PRODISCOVERY KIT.
WE JUST HAVE BEEN ABLE TO AWARD
TWO TEAMS ONE FROM HAWAI'I
WORKING ON A STANDARDIZED
STANDARDIZED METHOD TO IMPROVE
THE ABILITY TO UNDERSTAND
LYMPHATIC STRUCTURE AND FUNCTION
THROUGH MODELS.
WE HOPE THOSE WILL BE AVAILABLE
SO THERE'S A STANDARD TO WORK ON
THE STANDARD AND FUNCTION AND
DRUG REPURPOSING.
AND THAT OPPORTUNITY ALLOWED FOR
$8 MILLION IN FUNDING.
TO SHOW THE BREADTH WE'RE TRYING
TO COVER AND I'VE HAD THE
OPPORTUNITY IN LESS THAN A YEAR
THIS SHOWS OUR DIFFERENT
PORTFOLIO TOPIC AREAS.
WE SPRINT FOR WOMEN'S HEALTH,
OUR PROGRAM WHICH ARE THE MAIN
BRAMS LIKE DIAGNOSTICS AND IN
THERAPEUTICS, OUR SMALL BUSINESS
TOPICS AND THEN ALL OF THE
DIFFERENT LYMPHATIC HEALTH
INTEREST AREAS WE'RE TRYING TO
COVER.
YOU CAN SEE WE'RE TRYING TO FILL
IN THE SPACES AND I'D SAY WE'RE
NOT DOING NOT BAD JOBS SO FAR.
YOU'LL SEE WHAT WE'RE WORKING ON
IS ADDRESSING THE BARRIERS OF
LYMPHATIC DIAGNOSIS AND
TREATMENT.
ARPA-H IS INVESTED IN
ACCESSIBILITY AND AFFORDABILITY
AND INCLUSIVITY.
PART OF THE PROGRAMS LIGHT AND
GLIDE INCLUDE DISCOVERY DUO
PROGRAM.
WHAT'S THAT LOOK LIKE?
THAT MEANS EACH TEAM HAS BEEN
ASKED TO IDENTIFY AN EARLY
INVESTIGATOR FROM AN UNDER
REPRESENTED AREA AND ALSO A
PATIENT OR CAREGIVER AMBASSADOR.
TOGETHER THAT TEAM WILL
REPRESENT THE BIG TEAM AND PART
OF AN ACCESSIBILITY TASK FORCE
SIMILAR TO HOW YOU'RE WORKING ON
COMMITTEES TOO.
THE DISCOVERY DUO TEAM WILL BE
TASKED WITH DOING PATIENT VOICE
SECTIONS WITH RURAL AREAS,
ADMINISTRATORS, EVEN TALK TO ALL
OF YOU TOO.
THE IDEA IS THEY'RE GOING TO
COME BACK AND WE'RE ALL GOING TO
WORK AS A TEAM AGAIN LIGHT AND
GLIDE TOGETHER, ALL THE
PERFORMERS TOGETHER AND WORK ON
KEY PERFORMANCE EDUCATORS A
STRATEGIC ROAD MAP HOW WE GET
THE TECHNOLOGIES TO NOT SIT ON A
SHELF BUT BE USED ONE DAY.
THAT'S ALL I HAVE.
>> THANK YOU SO MUCH TO BOTH OF
YOU.
AGAIN WE'LL HAVE A PANEL
DISCUSSION AT THE END AND LOOK
FORWARD TOO A VIBRANT Q&A.
THE NEXT SPEAKER IS DR. MICHELLE
WASHKO THE DIRECTOR FOR NATIONAL
CENTER FOR HEALTH RESOURCES AND
SERVICES ADMINISTRATION.
>> GOOD MORNING.
I'M PLEASED TO BE HERE TODAY.
I'M DR. WASHKO FOR THE DIRECTOR
FOR WORKFORCE ANALYSIS IN THE
HEALTH RESOURCES AND SERVICES
ADMINISTRATION IN HHS.
THE MISSION IS TO IMPROVE HEALTH
OUTCOMES AND ACHIEVE HEALTH
EQUITY THROUGH ACCESS TO QUALITY
SERVICES, A SKILLED HEALTH
WORKFORCE AND INNOVATIVE HIGH
VALUE PROGRAMS.
MY AREAS OF EXPERTISE FOCUSES ON
THE HEALTH WORKFORCE.
THOSE WOULD RESEARCH AND TREAT
LYMPHATIC DISEASES.
TO THAT END, HRSA LOOKS TO GROW
THE HEALTH WORKFORCE IN THE
U.S. AND WHERE MY RESEARCH
CENTER SITS ADMINISTERS GRAND
LOAN AND SCHOLARSHIP PROGRAMS TO
THIS EFFECT.
WE USE OUR $2 BILLION ANNUAL
APPROPRIATION TO FUND EDUCATION
AND TRAINING PROGRAMS AT
UNIVERSITIES AROUND THE COUNTRY
THAT CREATE THE NEXT GENERATION
OF HEALTH CARE WORKERS.
WE USE THOSE GRANT PROGRAMS TO
FUND CONTINUING EDUCATION FOR
EXISTING HEALTH CARE WORKERS AND
ALSO SUPPORT COMMUNITY BASED
TRAINING AND ENCOURAGE CLINICIAN
TO WORK IN HIGH NEED AREAS.
MY REMARKS WILL HELP SET CONTEXT
AS TO WHY WE DO WHAT WE DO.
HIGH QUALITY HEALTH CARE STARTS
WITH A WELL-TRAINED ADEQUATELY
SUPPLIED AND DISTRIBUTED
WORKFORCE THUS OUR PROGRAM'S
AIMS TO DO THAT IN THE UNITED
STATES.
THERE ARE SEVERAL CHALLENGES IN
WORKFORCE SHORTAGES,
MALDISTRIBUTION AND UNEQUAL
ACCESS TO CARE AND EMPLOYMENT
QUALITY IN THE SECTOR.
THERE ARE PEOPLE ATTEMPTING TO
COMBAT THESE ISSUES.
DELVING DEEPER.
FIRST, THE SHORTAGES YOU CAN SEE
ARE IN MANY MANY OCCUPATIONS.
MY CENTER RELEASES WORKFORCE
PROJECTIONS ANNUALLY AND GETTING
STEADILY WORSE.
WE NEED TO BALANCE OUR SUPPLY
WITH DEMAND FOR CARE BY
EXPANDING THE NUMBER OF
INDIVIDUALS WHO WORK IN HEALTH
CARE.
SECOND, WHAT WE MAY PERCEIVE AS
SHORTAGES IN SMALLER GEOGRAPHIC
AREAS AND STATES, YOU ARAN
VERSUS RURAL COUNTIES AND THINGS
LIKE THAT THERE'S OFTEN THE
RESULT OF MALDISTRIBUTION ON A
TRUE SHORT AND.
WE NEED TO IMPROVE WORKFORCE
DISTRIBUTION BY ENCOURAGING
TRAINING AND THIS IS SOMETHING
THE PROGRAM ADMINISTER DO.
THIRD AND ACCESS TO CARE IS A
MUTHE PROBLEM IN THE COUNTRY.
WE NEED TO INCREASE AND IMPROVE
ACCESS TO MAKE IT EASIER FOR
PEOPLE TO GET THE HEALTH CARE
THEY NEED AND LABOR MARKET
FACTORS AND EMPLOYMENT
CHARACTERISTIC CAN DRIVE PEOPLE
OUT OF THE HEALTH CARE SECTOR
AND NEED TO IMPROVE THE QUALITY
OF EMPLOYMENT AND ADDRESS UNDER
COMPENSATION AND BURN OUT AND
WORKPLACE VIOLENCE.
TO GIVE A LITTLE BIT OF A FLAVOR
OF WHY WE'RE APPROPRIATED BY
CONGRESS TO FUND THESE GRANT,
LOAN AND SCHOLARSHIP PROGRAMS.
THESE ARE SHORTAGES AND THE
PROJECTION NUMBERS WE RELEASE ON
AN ANNUAL BASIS AND ABOUT TO
RELEASE NEW ONES AND UPDATE THEM
EVERY YEAR.
WE'RE LOOKING AT 30 OUT OF 35
SPECIALTIES PROJECTING TO HAVE A
SHORTAGE SOME OF THE WORSE WHERE
IN THIS CARE AND GERIATRICIANS
WILL CONTINUE TO EXPERIENCE SOME
THEREOF WORSE.
REGISTERED NURSES WE'RE
PROJECTING A 9% SHORTAGE BY
2026.
SO LESS OF A SHORT AND BUT IT
WILL CONTINUE AND SAME IN 9 OF
THE BEHAVIORAL HEALTH
OCCUPATIONS, PSYCHIATRISTS AND
OTHERS.
ANOTHER MAJOR CHALLENGE IS
DISTRIBUTION.
SOMETIMES WE MAY HAVE AN
APPLICATION WHICH APPEARS TO BE
AN EQUILIBRIUM AT THE NATIONAL
LEVEL BUT LOOKING AT THE SMALLER
GEOGRAPHIES THE MALDISTRIBUTION
LOOKS LIKE A SHORTAGE.
THAT SHORTAGE OF REGISTERED
NURSES EXAMPLE LOOK AT WHAT
WE'LL HAVE BY STATE IN 2036, 15
YEARS OUT YOU'LL SEE SOME OF THE
STATES IN DARK BLUE ARE
COMPLETELY ADEQUATE AN
OVERSUPPLY BUT THOSE IN DARK RED
WILL BE EXPERIENCING DIRE
SHORTAGES.
IT'S NOT THAT THE SHORTAGE OR
RATHER THE DISTRIBUTION IS EVEN
ACROSS THE STATES.
WE CAN EVEN IT OUT THE SHORTAGES
COULD BE MITIGATED.
THE THIRD THING WERE ACCESS
DIFFICULTIES.
AND THIS IS A GOOD EXAMPLE OF
WHAT THAT MEANS SHORTAGES CAN BE
FELT ACUTELY DEPENDING ON WHERE
SOMEONE LIVES.
THE EXAMPLE IS THE OUTLOOK FOR
WOMEN'S HEALTH SERVICE.
RIGHT NOW THEY'RE SEVERELY
HAMPERED DUE TO LACK OF ACCESS
TO PROVIDERS IN THE U.S.
WE'RE PROJECTING A 13% SHORTAGE
BY 2026 AND THE SHORTAGE IS
GOING TO JUMP TO 18%.
IT'S GETTING WORSE YEAR BY YEAR.
AS OF 2022, THIS IS TWO YEARS
AGO ALMOST 13% OF WOMEN OVER 18
LIVE IN COUNTIES WITH NO OB-GYN
PHYSICIANS.
NOT DIRECTLY RELATED TO
LYMPHATIC CARE AND DISEASE BUT
GIVES A SENSE OF WHERE WE ARE.
COUPLED WITH PRIMARY CARE
PROVIDER SHORTAGES THESE ARE
LACKING IN MANY PARTS OF THE
COUNTRY BECAUSE PRIMARY CARE
PROVIDERS ARE OFTEN PICKING UP
FOR OB-GYNs AND THIS HAPPENS
WHEN INDIVIDUALS ARE ABLE TO
WORK TO THE TOP OF THEIR
SCHEDULED PRACTICE OR ONE
PARTICULAR SPECIALTY GOES TO THE
NEXT.
THE LAST MAJOR CHALLENGE WAS
UNEVEN QUALITY IN THE U.S.
AND NOT ONLY THE QUALITY OF THE
WORKFORCE VIA TRAINING AND
EDUCATION BUT QUALITY OF
EMPLOYMENT CONDITIONS FOR HEALTH
CARE WORKERS.
A GREAT EXAMPLE ARE PRIMARY CARE
PHYSICIANS AGAIN.
THEY HAVE LOWER WAGES COMPARED
TO SPECIALTIES.
IT LEADS TO MANY MEDICAL SCHOOL
GRADUATES CHOOSING OTHER
EXPERIMENTS WHICH MEANS
PERSISTENT SHORTAGES AND WE ONLY
HAVE TWO GEOGRAPHIC LOCATIONS
AND STATES THAT WON'T BE IN
SHORTAGE OF PRIMARY CARE
PHYSICIANS IN THE NEXT 15 YEARS.
IMPROVED LABOR MARKET AND
EMPLOYMENT CONDITIONS ARE
CRITICAL PART OF THIS SOLUTION.
JUST BRIEFLY IN THE INTEREST OF
TIME, IT IS ONE THING OF COURSE
TO IDENTIFY THE CHALLENGES AND
TO UNDERSTAND HOW TO PROPERLY
ADDRESS AND IDENTIFY WHERE WITH
WE SEE SUCCESS AND UNDERSTAND
THE UNDERLYING CAUSES AND AGAIN
OUR PROGRAMS ATTEMPT TO MOVE THE
DIAL BY IMPACTING THE WORKFORCE.
UNFORTUNATELY, THOSE ARE TWO
MAJOR DRIVERS WHERE OUR FUNDING
CAN'T IMPACT.
SO THESE AREN'T THE ONLY ONES OF
COURSE BUT THIS IS MANY COULD GO
FROM A WORKFORCE.
FIRST THERE ARE MAJOR
POPULATIONS AFOOT IN THE
U.S. FOR SEVERAL GENERATIONS AND
THERE ARE FACTORS THAT PLAY A
ROLE AS A PROBLEM AND STLUGS.
-- SOLUTION.
THE POPULATION SHIFTS ARE THE
AGING OF THE POPULATION.
OUR AGING SOCIETY IS STILL THE
PRIMARY DRIVER FOR DEMAND GROWTH
IN HEALTH CARE.
WE HAVE ABOUT 17% OF THE
U.S. POPULATION OVER THE AGE OF
65.
BY 2050 THIS IS GOING TO JUMP TO
AROUND 23%.
THESE WILL BE COMPETING IN THE
WORKFORCE.
WE HAVE ATTRITION FROM THE
WORKFORCE WHILE DEMAND IS
INCREASING.
AT THE SAME TIME, U.S. BIRTH
RATE HAS FALLEN BY 20% SINCE
2007 AND INTERNATIONAL MIGRATION
ARE LOWEST AND CONTRIBUTE TO THE
LABOR SHORT AND AND INABILITY OF
THE HEALTH WORKFORCE TO MEET THE
GROWING HEALTH CARE DEMANDS
REQUIRED BY OUR POPULATION.
AND SECOND I MENTIONED LATE
LARGE LABOR MARKET FACTOR AND
CHALLENGES FACING ANYONE
ENTERING HEALTH CARE RESEARCH OR
WORKFORCE.
THERE'S ISSUES WITH EDUCATIONAL
PIPELINE FOR WORKERS AND NOT
ONLY SHORTAGES OF NURSES BUT
NURSING FACULTY WHO TRAIN THE
NURSES IN THE COUNTRY.
THIS MEANS LESS PEOPLE ARE ABLE
TO BE TRAINED AND LESS WORKERS
IN THE SUPPLY IN THE ANNUAL
BASIS.
UNDER COMPENSATION IS ALSO AN
ISSUE AND I MENTIONED PRIMARY
CARE AND THE DIRECT CARE
WORKERS.
IN THE LATTER CASE EASY ENTRY
APPLICATIONS CAN GET SIPHONED TO
OTHER INDUSTRIES.
THEY DON'T EVEN ENTER HEALTH
CARE OR DO AND IMMEDIATELY
LEAVE.
POOR WORKING ENVIRONMENT WITH
BURNOUT AND STRESS AND COVID-19
EXACERBATED THAT WHEN THERE WAS
LONG HOURS AND EXHAUSTION AND
EXPOSURE TO DEATH AND SUFFERING
AND PATIENT SAFETY.
AND OUR PROGRAMS DON'T TOUCH
DIRECTLY ON IMPACTING THESE
LARGE SOCIETAL ISSUES BUT DO
ATTEMPT TO PREPARE CLINICIANS TO
DEAL WITH THEM AND OTHER
PROFESSIONALS.
DESPITE ALL THESE CHALLENGES, I
KNOW I'M PAINT DIRE PICTURE BUT
THERE ARE SOLUTIONS WITHIN OUR
REACH.
WHILE WE CAN'T CHANGE THE
POPULATION DRIVERS WE CAN
PRODUCE MORE PROVIDERS TO GROW
THE NUMBER OF STUDENTS ENTERING
HEALTH CARE AND GRADUATING.
WHEN IT COMES TO DISTRIBUTION
AND AGAIN THIS IS SOMETHING OUR
SCHOLAR PROGRAM PROGRAMS DO
TOUCH ON THE CORPS BEING THE BIG
ONE AND ENCOURAGE THOSE TO
ENROLL IN UNDER SERVED AREAS
WITH LOAN REPAYMENT PROGRAMS WE
CAN CONTINUE TO ENCOURAGE TEAM
AND INTEGRATED CARE I MENTIONED
BEFORE.
WE CAN'T SOLVE ALL ACCESS
PROBLEMS WITH WORKFORCE
SOLUTIONS WE CAN ATTEMPT TO
DIVERSIFY AND OUR GRANTS DO THAT
TO HELP REACH UNDER SERVED
COMMUNITIES.
WE HAVE SEVERAL PROGRAMS THAT
SPECIFICALLY ATTEMPT TO
DIVERSIFY THE WORKFORCE BY
ATTRACTING VARIOUS KINDS OF
STUDENTS AND CAN CONTINUE TO
HARNESS AND USE TECHNOLOGY AND
TELEHEALTH AND A.I. AND LAST WE
CAN PROMOTE PROVIDER RESILIENCE
AND HEALTHY LABOR FORCE WHEN
EMPLOYERS INVEST IN LIVING WAGES
AND INVEST IN HEALTH AND
WELLNESS OF STAFF WE'VE SEEN
THIS REDUCE EMPLOYER COST AND
IMPROVE RETENTION AND IMPROVE
OUTCOMES IN THE WORKFORCE
RESILIENCY GRANTS.
SO JUST SOME PROMISING
DEVELOPMENTS YOU'VE SEEN AGAIN
ON THE FOUR AREAS, CHALLENGES IF
YOU WILL.
WE HAVE SEEN MEDICAL SCHOOL
ENROLLMENT INCREASE NEAR 6% IN
THE PAST FIVE YEARS.
SAME FOR REGISTERED NURSES
THEY'RE INCREASING BY 36%.
THE PROBLEM IS ON THE DEMAND
SIDE TOO.
WE HAVE DEMAND GOING UP WHILE
THESE INDIVIDUALS ARE BEING
ATTRACTED TO HEALTH CARE AND
WHAT THIS TELLS US IS THAT
PEOPLE ARE STILL INTERESTED IN
WORKING IN THIS SPACE.
NOT ONLY THAT WE'RE DIVERSIFYING
BY GENDER IN TERMS OF MEDICAL
SCHOOL STUDENTS.
WE HAVE SEEN GROWTH FOR WOMEN BY
14% BETWEEN 2019 AND 2024 WHILE
THE NUMBER OF MALE MEDICAL
STUDENTS DECREASED BY 3%.
TEAM BASED CARE FOLLOWING THE
TREND OF SEVERAL DECADES
SPECIALTIES ARE EXPECTED TO
EXPERIENCE A WORSENING SHORTAGE
BUT WE HAVE AN OVERSUPPLY OF
PHYSICIAN ASSISTANTS AND THAT
WILL TREND FOR MANY YEARS NOW.
WHAT WE'RE SEEING IS THAT THESE
ADVANCED PRACTICE PRACTITIONERS
ARE ACTUALLY ABLE TO HELP
MITIGATE SOME OF THE SHORTAGES
WE'RE SEEING WITH PHYSICIAN
SPECIALTIES.
WHILE PRIMARY CARE DOCTORS THE
SURPLUSES IN ADVANCED
PRACTITIONERS CAN ALLEVIATE
SHORTAGES BY MAKING UP FOR THE
DISTRIBUTIONAL IMBALANCES, IF
THAT MAKES SENSE.
ANOTHER SOLUTION I MENTIONED,
TECHNOLOGY.
I STARTED BY SAYING WE'RE
LOOKING AT SHORTAGES FOR MOST
BEHAVIORAL PROFESSIONS.
TELEHEALTH ROSE FROM 1% TO 40%
AND REMAIN ELEVATE.
THIS IS IMPORTANT BECAUSE YOU
CAN SEE OUR COLLEAGUES AT SAMHSA
AND HHS HAVE TRACKED WITH
NATIONAL SURVEY OF SUBSTANCE USE
AND HEALTH THAT NEARLY 6 MILLION
LITTLE OVER OF ADULTS ARE
REPORTING IN THE LAST SURVEY
SAID THEY ARE DID NOT RECEIVE
MENTAL HEALTH TREATMENT BECAUSE
THEY CAN'T ACCESS A HEALTH CARE
PROVIDER.
THIS IS HOW TECHNOLOGY CAN HELP
IMPROVE ACCESS.
THE LAST THING I'LL MENTION,
WE'VE SEEN PROMISING
DEVELOPMENTS IN PROVIDER
RESILIENCY.
MY COLLEAGUES AT THE AMERICAN
MEDICAL ASSOCIATION HAVE BEEN
TRACKING BURNOUT SINCE THE
PANDEMIC AND YOU CAN SEE WE HIT
A HIGH OF 62.8% OF PHYSICIANS IN
THE U.S. REPORTING AT LEAST ONE
SYMPTOM OF BURNOUT IN 2021 AND
THAT NUMBER HAS NOW STEADILY
FALLEN AND TIMELY FOR THE FIRST
TIME IT'S BELOW 50% SINCE THE
COVID-19 PANDEMIC.
YOU CAN SEE THIS IS A PERSISTENT
ISSUE.
THE PANDEMIC BROUGHT ATTENTION
TO IT BUT IT MEANS WE HAVE TO
KEEP INVESTING IN OUR WORKFORCE
BY TRAINING AND EDUCATING OUR UP
AND COMING CLINICIANS AS WELL AS
THOSE ALREADY EXISTING IN THE
LABOR FORCE TO MANAGE THEIR
RESILIENCY AND TO MANAGE BURNOUT
AND WELL BEING IN CONJUNCTION
WITH EMPLOYERS.
SO MY TWO TAKEAWAYS HERE WITH
FUNDING FOR THE WORKFORCE, WHILE
THERE ARE CHALLENGES AND THERE'S
NUMEROUS THAT FACE US AND THEY
ARE PERSISTENT AND THERE ARE
UNDERLYING FACTORS DRIVING THEM
THAT ARE QUITE LARGE, THERE ARE
SUCCESSFUL WAYS TO ADDRESS THEM
IF WE'RE WILLING TO CONTINUE TO
BE CREATIVE, OPPORTUNISTIC AND
TRY NEW STRATEGY.
THAT'S IT.
THANK YOU VERY MUCH.
>> THANK YOU, DR. WASHKO.
OUR NEXT SPEAKER REPRESENTS THE
CDC, DR. LISA RICHARDSON IS THE
DIRECTOR OF THE DIVISION OF
CANCER PREVENTION AND CONTROL.
>> GOOD MORNING, EVERYONE.
THANK YOU FOR THE INVITATION.
CAN YOU HEAR ME?
I ALWAYS FEEL LIKE I'M TALKING
INTO A VOID.
>> WE CAN HEAR YOU.
>> THANK YOU.
THANKS FOR HAVING ME.
THIS WILL BE A LIGHT TOUCH TODAY
FROM THE CDC.
OUR WORK HERE IS MOSTLY IN THE
INFECTIOUS DISEASE AREA.
AND WHAT HAPPENS WITH CANCER
PATIENTS.
THE FUNDING IN OUR DIVISION IS
ABOUT $355 MILLION A YEAR.
LYMPHEDEMA IS MOSTLY A CANCER
SURVIVOR ISSUE AND OUR FUNDING
IS ONLY ABOUT $750,000 OUT OF
THE ENTIRE BUDGET.
SO SINCE IT IS A GENERAL PROBLEM
ACROSS CANCER AND PEOPLE HAVE
SURGERY AND OTHER TREATMENTS, WE
HAVE ATTEMPTED TO DO SOME WORK
IN THAT AREA.
THIS IS KATHY BATES.
I MET HERE YEARS AGO.
ONE THING THE LYMPHATIC
EDUCATION RESEARCH NETWORK
HIGHLIGHT AND THEY HIGHLIGHT IT
EVERYWHERE IS THAT THE SEVERITY
THE OCCURRENCE OF LYMPHEDEMA AS
WELL AS THE SEVERITY AND IMPACT
ON QUALITY OF LIFE IS NOT
SOMETHING THAT IS GENERALLY
STUDIED.
A LOT OF PHYSICIANS AREN'T AWARE
AND PATIENTS AS WELL DON'T KNOW
WHAT TO LOOK FOR.
WHAT YOU SEE IS A VIDEO SHE DID
FOR US A YEAR AFTER WE MET BUT
THERE WAS SORT OF WHEN I MET
WERE LIKE, SO, WHAT CAN WE DO?
WE HAVE NO DEDICATED FUNDING FOR
LYMPHEDEMA.
WHAT WE DID DO AT THAT POINT WAS
CREATE A WEB RESOURCE
INFORMATION TARGETED TOWARD
CANCER SURVIVORS.
WHEN I LOOK AT THE DATA OF HOW
MANY PEOPLE COME TO OUR WEBSITE
AMONG OUR CANCER SURVIVOR
WEBSITE THIS IS ALWAYS IN THE
TOP THREE TO FIVE.
THERE SEEMS TO BE A HUNGER FOR
INFORMATION.
A COUPLE HUNDRED THOUSANDS OF
VISITS PER YEAR WHEN YOU THINK
OF THE CONTEXT OF WHAT'S OUT
THERE AND PROMOTE INFORMATION
AND THE CONSEQUENCES FOR
PATIENTS WHO HAVE THIS CONDITION
AND DON'T FEEL HEARD.
OUR ADVOCATES WENT TO CONGRESS
AND WERE ABLE TO SECURE SOME
FUNDING.
IT WASN'T GIVEN TO THE CANCER
CONTROL DIVISION.
MY BACKGROUND I FORGOT TO
MENTION IS IN MEDICAL ONCOLOGIST
AND WHEN I WAS PRACTICING I SAW
BREAST CANCER PATIENTS SO
LYMPHEDEMA IS SOMETHING WE SAW A
LOT AND CONGRESS FUNDED CHRONIC
DISEASE EDUCATION AWARENESS AND
MULTIPLE GROUPS WERE FUNDED
INCLUDE OBSTRUCTIVE SLEEP APNEA,
HEARING LOSS AND HAIR LOSS AND
COPD AND CYSTIC FIBROSIS.
IT'S A PLACE WHERE THE FORGOTTEN
AND RARE CONDITIONS THAT AREN'T
GETTING A LOT OF ATTENTION IN
THE PUBLIC HEALTH ARENA.
.
SOME ARGUE IS IT PUBLIC HEALTH
OR MEDICAL CARE?
WE STRUGGLE WITH THAT WHEN I'M
OUT TALKING ABOUT THE WORK WE
DO.
THIS WAS A GRANT NOT OUR USUAL
FUNDING MECHANISM WHICH IS A
COOPERATIVE AGREEMENT.
THIS ONE ENDED YESTERDAY AND THE
NEW ONE WILL BE COMING UP
TOMORROW.
JUST TO GO OVER A FEW OF THE
ACCOMPLISHMENTS OF THE FUNDING
WAS ABOUT $1.1 MILLION OVER
THREE YEARS.
THEY DID A TON OF WORK, CREATED
ONE-PAGE SUMMARIES AND NATIONAL
INDICATOR REPORT AVAILABLE ON
THEIR WEBSITE AND WE HAVE LINKS
FROM OUR WEBSITE AS WELL.
LOTS OF PRESENTATIONS,
CONFERENCES, SPEAKING
ENGAGEMENTS AND OTHER
OPPORTUNITIES.
SOW WHAT -- SO YOU SEE THE
REPORT AND ONE OF THE BIGGEST
CHALLENGES AND OTHERS HAS HAD AS
WELL IS WHAT IS THE PATIENT
EXPERIENCE IN THE FIELD OUT
WHERE YOU LIVE AND YOUR DAILY
LIFE.
AND THEY DID SURVEYS, ANALYZED
DATA AND PUBLISHED A PAPER AND
I'LL SHOW YOU ONE GRAPHIC FROM
THAT LATER.
AGAIN, THERE'S ASK THE EXPERTS.
SOME OF THE WORK THEY DID AND
THE INDICATOR REPORT WEB PAGE
HAS ACCESS TO THE REPORT,
DOWNLOADS, FEATURES AND OVER
1,000 HEALTH CARE PROVIDERS THAT
RECEIVED INFORMATION RELATED TO
LYMPHEDEMA.
I BELIEVE THIS IS THE LAST
SLIGHT.
AS I SAID EARLIER OUR WORK
WITHIN THE AREA OF LYMPHEDEMA
HAS NOT BEEN -- IS A CHALLENGE.
WE DO IT, CANCER SURVIVOR,
HEALTH AND WEALTHNESS, QUALITY
OF LIFE IS PRIORITIZED WITHIN
THE FUNDING LINE WE HAVE BUT
WHAT THE GRAPH SHOWS AND I'D
RECOMMEND YOU GET THE PAPER AND
READ IT.
IT'S NICELY DONE BUT HERE IT
TALKS ABOUT HAS ANYONE EVER
TALKED TO YOU ABOUT IT OR
EXAMINED YOU AND HAVE YOU EVER
BEEN REFERRED.
YOU'LL SEE THE VAST MAJORITY OF
PEOPLE THAT HAVE LYMPHEDEMA SAY
THESE HAVEN'T HAPPENED AND SOME
ARE AN EFFORT TO EDUCATE BOTH
SIDES OF THE COIN AND EDUCATE
PEOPLE WHO HAVE LYMPHEDEMA, AT
RISK FOR LYMPHEDEMA AND EDUCATE
PROVIDERS THAT WAY AS WELL.
AT CDC THAT'S ONE THING WE TRY
TO DO AND EDUCATE THE PEOPLE WHO
ARE AT RISK OR HAVE THE
CONDITION AS WELL AS THOSE
PROVIDERS WHO TAKE CARE OF THE
PATIENTS.
THIS HAS BEEN A GREAT
OPPORTUNITY FOR ME AND THANKS
AGAIN HEARING THE WORK OF THE
OTHER FEDERAL AGENCIES.
I SEE A LOT OF PLACES WHERE YOU
MIGHT BE ABLE TO MAKE
CONNECTIONS SIMILAR TO WHAT
DR. KENNEDY SAID ABOUT COMING TO
THE MEETING LAST TIME.
I SEE PLACES THAT OVERLAP HERE
AND THAT'S ALL I HAVE.
I LOOK FORWARD TO QUESTIONS AND
RECOMMENDATIONS AND SUGGESTIONS
FROM THE GROUP.
THANK YOU.
>> THANK YOU, DR. RICHARDSON.
OUR FINAL SPEAKERS ARE FROM THE
FDA, WE HAVE DR. JORDAN POMEROY
SENIOR PHYSICIAN IN THE DIVISION
OF CARDIOLOGY AND DR. SONIA
SINGH THE CLINICAL REVIEWER FOR
THE DIVISION OF ONCOLOGY TOO.
>> I'LL SEE IF MY COLLEAGUE,
DR. SINGH IS ON THE LINE.
>> GOOD MORNING.
I'M THE LINE.
JORDAN, WOULD YOU LIKE TO
INTRODUCE YOURSELF FIRST.
>> SOUNDS GOOD.
THANKS FOR THE INVITATION ON
BEHALF OF FDA TO INTRODUCE THE
COMMISSION.
I'M JORDAN POMEROY I'M A
CLINICAL REVIEWER MEDICAL
OFFICER WITHIN THE DIVISION OF
CARDIOLOGY AND NEPHROLOGY AND
CARDIOLOGIST BY TRAINING IN
GENERAL AND IT'S A LYMPHATIC
ORIGIN OF THE DISEASE GOING TO
THE DIVISION OF CARDIOLOGY AND
NEPHROLOGY AND EXTREME
CARDIOVASCULAR DISEASES AND
ALLOW DR. SINGH TO INTRODUCE
HERSELF.
>> GOOD MORNING.
I'M A PEDIATRIC ONCOLOGIST AND
CLINICAL REVIEWER AT THE FDA.
THANK YOU FOR INVITING US TO
PARTICIPATE IN THE MEETING AND
HAPPY TO JOIN THE DISCUSSION.
I WAS GOING TO BRIEFLY INTRODUCE
FDA ITSELF, JORDAN, UNLESS YOU
HAD PLANS TO DO SO I CAN TALK A
LITTLE BIT ABOUT HOW WE OPERATE.
>> ABSOLUTELY.
HAPPY TO FOLLOW UP.
>> GREAT.
THE FDA HAS SEVERAL CENTERS.
I OPERATE IN THE CENTER OF DRUG
EVALUATION AND RESEARCH AS WELL
AS JORDAN.
AND IN THAT OFFICE I WORK WITHIN
OOD OR THE OFFICE OF ONCOLOGIC
DISEASES I REVIEW DRUG
DEVELOPMENT PROGRAMS WITH
PEDIATRIC SOLID AND RARE AND
BRAIN TUMORS SPECIFICALLY WITHIN
THE DIVISION OF ONCOLOGY TOO.
I DON'T HAVE SLIDES BUT WOULD
LIKE TO SELL THE GROUP ABOUT OOD
FOR ANYONE NOT FAMILIAR.
THIS OFFICE OVERSEES THE
DEVELOPMENT, APPROVAL AND
REGULATION OF DRUGS AND
BIOLOGICS FOR CANCER.
WE'RE CHARGED WITH ENSURING THE
THERAPIES ARE SAFE AND EFFECTIVE
FOR THE USE OF THE U.S. PUBLIC
AND FOR CONTEXT OOD CONSISTS OF
MORE THAN 130 PHYSICIANS,
SCIENTISTS AS WELL AS REGULATORY
PROJECT MANAGERS.
OUR GROUP HAS EXPERTISE IN SOLID
TUMORS AND MAG -- MALIGNANCIES
AND THREE DIVISIONS.
ONE IS SOLID TUMORS AND TWO
DEDICATED TO HEMATOLOGIC
MALIGNANCIES AND FOR THE
NON-CLINICAL REVIEW TEAM
OVERSEEING PROJECTS.
IN TOTAL WE COMPRISE SIX
DIFFERENT DIVISIONS AND OUR
OFFICE WORKS IN PARTNERSHIP WITH
THE ONCOLOGY CENTER OF
EXCELLENCE OR THE OCE.
SOME MAY BE FAMILIAR WITH THIS.
THIS BODY IS HEADED BY
DR. CANISTER AND THE OCD SERVES
AS AN INTER-CENTER INSTITUTE
DESIGN TO HELP STREAMLINE REVIEW
AND INITIATIVES AND HELPS TO
LEAD RESEARCH, EDUCATIONAL
OUTREACH PROJECTS AND OTHER
PROGRAMS FOR DRUG DEVELOPMENT
FOR PATIENTS WITH CANCER.
I KNOW WE'RE SHORT ON TIME SO I
WON'T GET INTO SPECIFICS OF THE
REGULATORY INITIATIVES DURING
THE INTRODUCTORY PORTION BUT I'M
SURE WE CAN MENTION THAT DURING
THE PANEL DISCUSSION SINCE THE
FDA HAS SEVERAL PROGRAMS TO
EXPEDITE DRUG DEVELOPMENT AS
WELL AS INITIATIVES TO PROMOTE
DRUG DEVELOPMENT FOR RARE
DISEASES.
I'LL STOP THERE AND JORDAN, FEEL
FREE TO ANYTHING YOU'D LIKE TO
ADD ABOUT YOUR DIVISION
SPECIFICALLY OR ANYTHING ELSE
YOU DO.
>> APPRECIATE THAT.
I AGREE WITH DR. SINGH.
I THINK OUR CONTRIBUTION IS BEST
IN THE PARAMETER OF DISCUSSIONS
AND QUESTIONS THAT MANY CO UP.
WE'RE THE ULTIMATE ORGANIZATION
THAT REVIEWS SAFE AND EFFECTIVE
MARKETING OF A DRUG VERSUS
DISEASE.
I THINK WHAT'S KEY HERE IS
LYMPHATIC DISEASES REPRESENT
UNMET CLINICAL NEED WITH A BAR
OF FLEXIBILITY WHEN WE CONSIDER
HOW TO REVIEW AN EVIDENTIARY
BASIS FOR REVIEWING SOMETHING IS
SAFE AND EFFECTIVE FOR USED FOR
SPECIFIC INDICATION.
WHILE WE'RE ON THAT PENULTIMATE
ORGANIZATION WE SEEK OUT EARLY
ENGAGEMENT.
AS DR. SINGH ELUCIDATED WE HAVE
SEVERAL MECHANISMS BY WHICH WE
CAN ENCOURAGE THAT EARLY
ENGAGEMENT BEEN THE PRECLINICAL
DEVELOPMENT SPACE AND HAPPY TO
DISCUSS THOSE AS QUESTIONS COME
UP.
I BELIEVE THE ONCOLOGY GROUP IS
A LARGER GROUP FOCUSSED ON AREAS
THAT ENCOMPASS LYMPHATIC DISEASE
MORE THAN VASCULAR AND WE DON'T
LEARN A LOT ABOUT LYMPHATIC
DISEASE IN OUR CLINICAL TRAINING
OTHER THAN A CARDIOLOGIST I
RECOGNIZE THE LYMPH SYSTEM
OUTLET IS CLOSE TO THE RIGHT
ATRIUM BUT EVEN THAT I THINK IS
NOT COMPLETELY UNDERSTAND HOW
THAT CIRCULATION WORKS BUT AGAIN
WE'RE HAPPY TO BE HERE TO
PARTICIPATE IN THE DISCUSSION
AND WE'D LOVE TO HEAR YOUR INPUT
AND HOW WE MAY BEST SERVE THE
COMMUNITY.
>> THANKS TO BOTH OF YOU AND
I'LL TURN IT OVER TO MY
CO-CHAIR.
>> WELL, THREW TO ALL OF OUR
PANELISTS AND GUESTS TODAY FOR
SHARING SO MUCH ABOUT WHAT YOU
DO.
YOU'VE INSPIRED A LOT OF
QUESTIONS EVEN COMING INTO THE
MEETING BUT I'M SURE MORE TODAY.
ALL OF OUR COMMITTEES HAVE
ALIGNED ON SOME QUESTIONS FOR
YOU SO I FIRST WANT TO TURN IT
OVER TO THE COMMITTEES TO ASK
THE QUESTIONS.
ANY VOLUNTEERS TO GO FIRST?
>> I REPRESENT THE MEASURABLE
RESULTS.
AND I JUST THINK I'M CURIOUS LOU
TO MEASURE HOW WELL THINGS ARE
WORKING.
METRICS ARE IMPORTANT TO AN
ORGANIZATION AND HOW THOSE
AFFECT YOUR PRIORITIES.
LET ME GIVE AN EXAMPLE.
WHAT IS THE LYMPHATIC DISEASE
COMMUNITY NEED TO DO TO GET
INSURERS TO HELP WITH LYMPHATIC
DISEASE?
THAT'S AN EXAMPLE.
HOW DO YOU MEASURE YOUR SUCCESS
IN DOING SOMETHING LIKE THAT?
IS IT WHAT YOU FUND AND HOW MUCH
YOU FUND?
WHERE YOU GO?
I'LL LET KIMBERLY ANSWER MORE
SFIFKLY BUT THERE'S SOME NICE
EXAMPLES OF PLAQUE MET RUKZ WE
PUT IN PLACE IN THE PROGRAM TO
REALLY DEFINE WHAT A THERAPEUTIC
LOOKS LIKES IN THIS SPACE.
THAT WAS HARD TO DO STARTING
FROM ZERO.
SO KIMBERLY DID A NICE JOB OF
THAT AND LET YOU SPEAK TO THAT
BUT METRICS ARE REALLY IMPORTANT
TO US.
I HAD TO PUT IN QUANTITATIVE HET
METRICS BASED ON EVIDENCE AND
A
AAS -- AND AS AMY SAID THERE'S
NOT A LOT OF EVIDENT.
WE HAD TO LOOK AT EXAMPLES CLOSE
TO LYMPHATICS AND/OR REACH OUT
TO STAKEHOLDERS FOR ADVICE BUT
THEN WE CREATED BASED ON AND
HAVE AT LEAST MINIMAL RESULTS OR
HOPEFULLY STRIVE FOR IDEAL.
WE MADE THEM DIFFICULT BUT
ACHIEVABLE GIVEN THE RESOURCES
TO GET IT DONE.
WHAT WE DID DO IS REACH OUT TO
FDA COLLEAGUES TO ENSURE IT WAS
DOABLE AND THEN WHAT WE DID WAS
WE BASED ON THE FDA GUIDELINES
AND ASKED OUR PERFORMERS TO COME
IN, OUR PROPOSERS TO COME IN AND
IF THEY CAN'T REACH THOSE
METRICS, THEY NEED TO BE ABLE TO
JUSTIFY TO US WHAT THEY BELIEVE
THEY CAN DO AND WE WOULD DISCUSS
AND NEGOTIATE.
BUT THE BUT IS THERE ARE DEFINED
METRICS.
THERE'S THREE PHASES IN OUR
PROGRAM AND IN ORDER TO MOVE TO
THE NEXT PHASE, THOSE METRICS
NEED TO BE COMPLETED,
DELIVERABLES DONE AND IF THERE'S
A LOT OF MANAGEMENT AND
OVERSIGHT THEN WE SIT TOGETHER
AND TRY TO FIGURE OUT WHY IT'S
NOT BEING DONE AND HOW WE COULD
MITIGATE THEM.
AND I THINK ANOTHER REALLY
IMPORTANT THING OUR FDA
COLLEAGUES HAVE MENTIONED,
ENGAGING EARLY.
SO WE ARE GOING TO ASK OUR
PROPOSERS, PERFORMERS TO ENGAGE
VERY EARLY AND WE'RE GOING TO
INVITE FDA IN AND NIH.
WE WANT EVERYONE AT THE TABLE.
SO THE TECHNOLOGIES ARE
SUCCESSFUL.
>> CAN I ASK A FOLLOW-UP
QUESTION?
SO, IS ARPA-H ARE YOU ABLE TO --
WOULD YOU BE ABLE TO SHARE WHAT
THOSE METRICS ARE WITH US?
THERE'S SPECIFIC CURIOSITY
AROUND THAT AND A BIGGER
QUESTION IS FOR ARPA-H AT THE
CONCLUSION OF THE SIX YEAR
PROGRAM HOW DO YOU DECIDE THIS
WAS A GOOD BET?
>> WE CAN SHARE THAT IT'S PUBLIC
AND KIMBERLY CAN SHARE THE PDF
VERSION.
WE DON'T HAVE SPECIFIC MELT
METRICS YET AROUND UP DARPA WE
DON'T HAVE THAT IN THE HEALTH
CARE SECTOR AND IN MANY CASES WE
NEED COMMERCIAL ENTITIES AND
HAND THEM OFF TO HEALTH CARE
PROVIDERS.
WE WANT TO MAKE SURE WE DON'T
MEET BILLION, MILLION-DOLLAR
THERAPIES AND HAVE COST METRICS
AND BUILD IN NEGOTIATING TERMS
IN OUR CONTRACTS REQUIRING COST
LIMITS.
THAT'S BUILT IN TO OUR
AGREEMENTS.
FOR A PROGRAM LIKE LIGHT AND
GLIDE IT'S EARLY THE END GOAL
MAY NOT BE SOMETHING FULLY
COMMERCIALIZABLE BECAUSE WE'RE
STARTING AT ZERO IN MANY CASES.
AND SOME MAY BE WILDLY
SUCCESSFUL AND COMMERCIALIZE AND
LICENSE OR MAKE A PRODUCT AT THE
END OF THE EFFORT OR IT MAY BE
SOME OF OUR GROUPS MAKE GREAT
SCIENTIFIC ADVANCES AND GO ON TO
BE OTHER MECHANISMS ACROSS THE
GOVERNMENT.
WHERE THAT LIVES FOR LYMPHATICS
IS DIFFICULT.
WE'RE HOPEFUL IN THE NEXT SIX
YEARS TO SHOW WHAT IS POSSIBLE
AND THERE WILL BE OTHER
OPPORTUNITIES.
>> SORRY, FOR CECELIA, BEYOND
PUBLICATIONS WHICH IS A VERY NIH
TYPE, HOW DOES MEDICAL
RESEARCH -- HOW DID DHA AND DAD
THINK OF THE SUCCESS AND OUTPUTS
OF THE CDMRP PROGRAM.
HOW DO YOU MEASURE SUCCESS AND
PROGRESS?
>> I THINK RETURN ON INVESTMENT
IS SOMETHING EVERY LARGE FUNDER
GOVERNMENT OR NOT GRAPPLES WITH.
IT'S VERY DIFFICULT FOR US TO
ASSESS.
FOR US WE'RE FAST PACED AND HAVE
HEIGHTENED POLITICAL AWARENESS.
WHAT WE MEASURE MOST IS HOW MUCH
WE INVEST THAT BECAME A
COMMERCIALIZED PRODUCT.
WE LOOKED AT THE APPROPRIATIONS
WE RECEIVED BEING OVER $1.5
BILLION THROUGH THE 30 YEARS OF
PROGRAM ON A DEEP DIVE AND WERE
ABLE TO DETERMINE HOW MANY
PRODUCTS AND WHAT WAS OUR
CONTRIBUTION TO THE SUCCESS.
THAT SERVED AS A VERY USEFUL
EXERCISE.
WE WERE ABLE TO SEE THE FUNDING
VEHICLES TO PRODUCE PRODUCTS.
AND THAT HAS LED INTO A HUGE
UNDERTAKING THROUGHOUT THE
ORGANIZATION.
HOW CAN WE BE MORE EFFECTIVE AND
FUND A PATENT OR IND FOR LESS
RESOURCES AND GET MORE BANG FOR
YOUR BUCK?
THAT'S WHAT WE'RE LOOKING AT.
OUR ADVOCACY GROUPS ASK HOW DID
YOU CONTRIBUTE, WHAT TYPE OF
PRODUCT AND ABOUT WHAT IS
SPECIALIZED FOR THE PROGRAMS AND
NOW WE ARE AT A POINT WHERE WE
HAVE A LOT OF FAST DATA AND WE
CAN SEE HOW SUCCESSFUL WERE WE
AND WITH THAT WE START SEEING
TRENDS OF WHICH AWARD MECHANISM,
WHICH FUNDING VEHICLE IS GOING
TO GENERATE BETTER RESULTS.
>> I SUPPORT ARPA-H.
I WAS CURIOUS ABOUT THE
ANNOUNCEMENT MECHANISM.
THIS I NEVER HEARD BEFORE.
I KNOW MANY COLLEAGUES AND I
THINK THIS TYPE OF PROGRAM IS
IDEAL FOR MORE PEOPLE IN THE
FIELD AND DIVERSE KNOWLEDGE BUT
I DON'T KNOW WHAT IS THE
MECHANISM YOU SEND THESE
ANNOUNCEMENTS.
HOW DOES IT WORK?
HOW DO YOU SEND IT?
>> SO EVERYTHING IS ANNOUNCED
PUBLICALLY.
ALL CALLS FOR PROPOSALS.
OUR GLIDE PROGRAM IS JUST
STARTING.
SO TOMORROW IS THE FIRST DAY SO
YOU'RE NOT TOO LATE.
IT'S VIRTUAL.
I DON'T EXPECT US TO FLY WITH US
IF YOU DON'T WANT TO BUT WE HAVE
A VIRTUAL PROPOSAL TO HEAR
DIRECTLY FROM KIMBERLY WHAT SHE
WANTS IN HER PROGRAM.
THERE'S OPPORTUNITIES FOR PEOPLE
AND IT LAUNCHES TOMORROW.
THAT PROGRAM IS NEWLY LAUNCHES
AND I'M SURE WE CAN PROBABLY
SEND IT OUT IN REAL TIME RIGHT
NOW THE INFORMATION RELATED TO
IT BUT ALL OF OUR ANNOUNCEMENTS
ARE MADE THROUGH OUR WEBSITE
THROUGH SAM DOT-GOV.
I ADMIT MANY ENT DO KNOW ABOUT
ARPA-H OR THE MODEL AND TRYING
OUR BEST AND GO OUT AND TRY TO
ATTEND AS MANY MEETINGS AS
POSSIBLE TO MAKE PEOPLE AWARE OF
THE FACT WE EXIST.
I WILL SAY KIMBERLY AND HER TEAM
HAVE DONE A FANTASTIC JOB TRYING
TO MAKE THE FIELD AS AWARE AS
POSSIBLE BUT AGAIN I'LL AGREE
100% WE NEED HETEROGENEOUS TEAMS
TO SOLVE THE PROBLEMS AND IT'S
RARE IN ARPA-H YOU HAVE ONE P.I.
ON A GRANT.
THEY'RE ON A COOPERATIVE
AGREEMENT OR T32 OR LARGE
CONSORTIUMS OF 10 TEAM MEMBERS
OR MORE WORKING TO SOLVE THE
PROBLEM.
THAT'S WHAT WE'RE AIMING TO DO
WITH OUR PROPOSERS DAY AND HAVE
PEOPLE COME IN AND TELL PEOPLE
WHAT THEY WANT AND MOST IN THE
AUDIENCE WILL SAY WE CAN'T DO
THAT BUT AND WE KNOW THAT BUT IF
YOU GET 10 PEOPLE WITH DIFFERENT
EXPERTISE CAN YOU DO THIS SO
IT'S OUR ATTEMPT TO REACH OUT.
THE PEOPLE THAT WILL BE IN
ATTENDANCE TOMORROW ARE THE CORE
TEAMS AWARE OF WHAT WE'RE DOING
AND THEY'LL GO OUT IN THE
COMMUNITY LOOKING FOR OTHER
RESEARCHERS TO HELP ROUND OUT
THEIR TEAM AND HOPING TO BUILD A
NETWORK.
WE WOULD LOVE AND APPRECIATE ANY
HELP WE CAN TO GET INFORMATION
OUT BUT RELEASED ON SAM DOT GOT
WHERE WE SOLICIT.
>> [INAUDIBLE]
>> CAN I JUST JUMP IN REALLY
QUICKLY.
I THINK WHAT HAS BEEN SO
ENLIGHTENING FOR ME AS I GO OUT
TO SPEAK WITH PEOPLE OUTSIDE THE
LYMPHATIC COMMUNITY, AS WE TALK
ABOUT LYMPHATICS, LIKE A LIGHT
BULB GOES OFF.
MAYBE WE CAN TAKE OUR ENABLING
TECHNOLOGY AND PUT IT TOWARDS
THE LYMPHATIC SYSTEM AND IT'S
BEEN SO EXCITING.
WE NEED EVERYONE'S HELP TO GET
THE WORD OUT.
WE HAVE DONE THE BEST WE CAN TO
GET THE WORD OUT BUT ANY HELP IS
LIKE, YES.
BECAUSE AS WE TALK TO PEOPLE, SO
MANY ARE SAYING WELL, THIS IS SO
COOL.
WHY ARE WE NOT THINKING ABOUT
THE LYMPHATIC SYSTEM.
IT'S SO IMPORTANT.
IT'S BEEN HUGELY MOTIVATING TO
KNOW THAT THERE ARE PEOPLE OUT
THERE THAT WANT TO BRING THE
TECHNOLOGY IN.
THE OTHER THING I WOULD SAY IS
ON THE PROGRAM'S WEB PAGE, SO
EACH PROGRAM HAS A WEB PAGE,
THERE'S A MECHANISM CALLED THE
TEAMING PAGE WHERE YOU CAN PUT
DOWN YOUR TECHNOLOGY, YOUR
EXPERTISE AND THAT'S A WAY THAT
PEOPLE CAN REACH OUT AND FIND
TEAMS BUT THEN AGAIN THAT'S THE
OPPORTUNITY WITH ARPA-H IS WE'RE
LOOKING FOR TEAMS.
WE WANT PEOPLE TO COME IN
BECAUSE WE KNOW THIS IS REALLY
HARD AND WE NEED ALL HANDS ON
DECK.
>> THANK YOU SO MUCH.
SO MAYBE CONTINUING ON THE THEME
OF GETTING THE WORD OUT AND
GENERATING BETTER AWARENESS WE
HAD A NUMBER OF QUESTIONS FROM
OUR EDUCATIONAL IMPERATIVE
COMMITTEE.
WOULD SOMEONE LIKE TO ASK ONE OF
THESE QUESTIONS?
>> COULD YOU REPEAT THE
QUESTION?
I DIDN'T HEAR THE LAST PART.
>> LET ME STATE THE QUESTION.
WHAT EDUCATIONAL INITIATIVES
HAVE YOU FOUND TO BE MOST
SUCCESSFUL WITH PATIENTS,
RESEARCHERS, PHYSICIANS, ALL
STAKEHOLDERS FOR YOUR
ORGANIZATION?
>> I CAN START, IF YOU DON'T
MIND.
THIS IS DR. RICHARDSON FROM CDC.
ONE THING WE NOTICED WHEN WE
DEVELOP OUR CAMPAIGNS WE DO OR I
DON'T REALLY LIKE CAMPAIGNS BUT
CONGRESSES DO THEM SO WE DO
THEM.
WHAT WE TRY TO DO IS TRY TO GO
INTO THE COMMUNITY OF THE PEOPLE
IMPACTED BY THE THINK WE'VE BEEN
ASKED TO WORK ON TO SEE WHAT
THEIR EXPERIENCING IN WHATEVER
AREA IT IS.
FOR INSTANCE, LET'S SAY WE WERE
ABLE TO DO SOMETHING WITH
LYMPHEDEMA EDUCATION MORE
PURPOSEFUL THAN WHAT WE'RE DOING
NOW BUT IT'S ABOUT GOING IN
COMMUNITIES AND TALKING WITH
PEOPLE WHO HAVE IT AND THEIR
CAREGIVERS AS WELL.
THAT'S THE HIDDEN POPULATION
NOBODY EVER TALKS TO AND
DR. KENNEDY TALKS TO YOU AND
THAT'S HOW WE HANDLE IT AND LIKE
WHY DON'T OUR MESSAGES WORK AND
HOW DO WE BETTER RESONATE BETTER
WITH THE PUBLIC AND PROVIDERS.
AND WE DON'T TALK TO THE PEOPLE
IMPACTED BY THE THING WE WANTED
TO HELP WITH.
THAT'S WHAT WE TRY TO DO AT
INCREDIBLE AS MUCH AS WE CAN AND
EVALUATE WHAT YOU DO TO THE
POINT THE QUESTION BEFORE I WAS
GOING TO IT RAISE MY HAND, WE
DON'T THROW INFORMATION LIKELOO
LIKE JELLO ON A WALL AND WE TRY
TO EVALUATE WHETHER IT WORKED OR
NOT BECAUSE IF IT DOESN'T WORK
YOU NEED TO STOP DOING IT AND DO
SOMETHING ELSE.
>> I'LL DOUBLE DOWN ON WHAT LISA
SAYS.
WE HAVE A VERY ROBUST
PERFORMANCE MEASUREMENT AND
SYSTEM FOR OUR BILLIONS IN
EDUCATIONAL AND TRAINING GRANTS.
WHAT WE HAVE ALSO FOUND MOST
IMPACTFUL IS WHEN YOU ARE
TRAINING HEALTH CARE WORKERS,
WRIT LARGE IN THE COMMUNITY.
IN THE COMMUNITIES, TALKING TO
PEOPLE IN THIS CASE IF YOU'RE
TALKING ABOUT LYMPHATICS, GO TO
THE PLACES AND WHERE YOU'LL FIND
PROVIDERS AND WHEN YOU ARE
TRAINING THOSE PROVIDERS AS
RESEARCHERS IN THOSE
COMMUNITIES, THEY ARE MORE
LIKELY TO STAY THERE SO THAT'S
IMPORTANT TO REMEMBER.
>> THIS IS JORDAN POMEROY FROM
THE FDA.
WHILE NOT COMPLETELY ON TOPIC
WITH THE EDUCATION PIECE I THINK
IT'S IMPORTANT FROM OUR
PERSPECTIVE AS WE THINK ABOUT
GENERATION OF SUBSTANTIAL
EVIDENCE AND EFFECTIVENESS OF
THE REGULATORY SPACE OFTEN TIMES
FOR LYMPHATIC DISEASES MAY NOT
BE CLEAR CLINICAL OUTCOMES THAT
CAN BE USED FOR DEVELOPING
REGULATORY STANDARD
UNDERSTANDING SUBSTANTIAL ITEMS
AND WE RECOMMEND YOU LISTEN TO
THOSE COMMUNITY TO DEVELOP AN
UNDERSTANDING OF WHAT'S
IMPORTANT FROM A SYMPTOMATIC AND
FUNCTIONAL BENEFITS.
AT SOME POINT WE'RE LIKELY TO
ASK YOU TO QUANTIFY THAT
IMPORTANCE IN A WAY THROUGH A
PATIENT-REPORTED OUTCOME OR
CLINICAL OUTCOME ASSESSMENT SO
WE CAN HAVE DATA BY WHICH TO
TEASE OUR REGULATORY STANDARD.
NOT ONLY EDUCATING THE PROVIDERS
BUT PROVIDING THE TOOLS.
>> TO PIGGY BACK OFF WHAT JORDAN
MENTIONED, THE FDA AS FAR AS A
PROGRAM FOR PATIENT-FOCUSSED
DRUG DEVELOPMENT.
I ENCOURAGE ANYONE INTERESTED IN
THE THINGS HE MENTIONED SUCH AS
CLINICAL OUTCOME ASSESSMENTS WE
CAN OFFER PATIENT LISTENING
SESSIONS DESIGN FOR SMALL
PATIENT POPULATION TO FIND OUT
WHAT'S OF IMPORTANCE IN TERMS OF
CLINICAL BENEFIT FOR THEM AND
HOW DO YOU MEASURE THAT IN A
DEVELOPMENT PROGRAM.
THERE ARE OTHER OPPORTUNITIES AS
WELL SO IT'S ALWAYS A RESEARCH
COMMUNITY.
>> THAT'S EXACTLY WHAT I WAS
GOING TO ASK.
GIVEN LYMPHEDEMA IS THOUGHT OF
AS INCREASED VOLUME BUT THE
SYMPTOMS ARE NOT RELATED TO THAT
AND DOES THE FDA CONSIDER
PATIENT-REPORTED OUTCOMES IN ITS
DECISIONS OF APPROVING NEW DRUGS
GIVEN WE DON'T HAVE VERY GOOD
TOOLS AND MEASURING DIRECTLY THE
LYMPHATIC FUNCTION.
>> SO, YES, DEFINITELY.
PATIENT-REPORTED OUTCOMES HAVE
PRECEDENT FOR RECT --
REGULATORY APPROVAL AND IT'S
IMPORTANT THROUGH THE
QUANTIFICATION WE UNDERSTAND
WHAT THE CLINICAL MEANINGFULNESS
OF ANY DESCRIBED CHANGE OR
SCORING TOOL YOU DEVELOP.
FOR RARE DISEASE, WHICH WE
DEFINE AS LESS THAN 200,000
CASES REPORTED PER YEAR, WE
DON'T FURTHER STRATIFY IT DOWN
TO ULTRA RARE BUT IN THAT SPACE
OFTEN TIMES THERE'S NOT A REAL
WEALTH OF INFORMATION ABOUT
CLINICAL OUTCOMES OR SOMETHING
RESOLVED IN A TEMPORALLY SHORT
MANNER AND EARLY ENGAGEMENT IS
IMPORTANT BECAUSE OFTEN YOU HAVE
TO PROVIDER VALIDATION OF THE
FIT FOR PURPOSE AND THAT OF
COURSE COMES WITH RISK AND SO WE
HAVE BOTH IN PATIENT FOR DRUG
DEVELOPMENT AND CLINICAL OUTCOME
ASSESSMENT TEAMS WITHIN THE FDA
AND OFFICE OF NEW DRUGS EXPERTS
IN THE DEVELOPMENT OF THESE
TOOLS WE ENGAGE THEM AT THE TIME
WE'RE OF ENGAGING TO ASSIST WIT
THE DEVELOPMENT OF IT.
>> DO THOSE FOLKS PUBLIC
GUIDELINES ON WHAT THEY CONSIDER
A MEANS OF LOOKING AT THIS OFFER
QUANTIFYING.
>> ANY BACKGROUND IS IN ONCOLOGY
AND NOT A SPECIALIST IN RARE
DISEASES LIKE THE REST OF THE
GROUP BUT I WOULD AND THIS IS
FOR THE OVERGROWTH SPECTRUM
WHICH CAN HAVE A COMPONENT OF
LYMPHATIC MALFORMATION.
WE'VE BEEN TALKING ABOUT
LYMPHEDEMA BUT WITH RESPECT TO
LYMPHATIC DISEASE AS A WHOLE
THERE'S HETEROGENEITY AND HOW DO
YOU MEASURE AND WHAT'S
IMPORTANT.
TO DOUBLE DOWN ON WHAT JORDAN
WAS MENTIONING IT'S IMPORTANT TO
ENGAGE WITH THE REVIEW DIVISION
EARLY AND OFTEN.
WHAT WE WILL CONSIDER IN OUR
DISCUSSIONS WITH SPONSORS AND
INVESTIGATORS INCLUDE THINGS
SUCH AS CLINICAL CONTEXT AND
NATURAL HISTORY OF THE DISEASE
AND WHAT ARE THE AVAILABLE
THERAPIES, ETCETERA.
THE MAIN MESSAGE WOULD BE FOR
SMALL PATIENT POPULATION ARE
DEALING WITH COMPLEX OR ATYPICAL
GROWTH PATTERNS TO ENGAGE EARLY
FOR THE PROGRAM I MENTIONED.
I'VE WORKED WITH THE DRUG
DEVELOPMENT FOR PATIENTS WITH
PROSE AND UP IN THAT POPULATION
WITH RESPECT TO PATIENTS WE HAD
ADDITIONAL DATA IN PHYSICIAN
REPORTED OUTCOMES WHICH WAS
HELPFUL IN CORROBORATING WHAT WE
WERE SEEING IN LESION SHRINKAGE.
THERE WAS FLEXIBILITY EXERCISE
IN THE PRIMARY END POINT GIVEN
THE COMPLEXITIES GIVEN AND THE
FACT PROSE IS A AHISTO
LOGICALLY BENIGN PRODUCTION AND
LOOKED AT THE MESHABLE --
MEASURABLE LESIONS AND IT WAS
ESTABLISHED BEFORE THEY PURSUED
THEIR PROGRAM AND WE ALSO HAD IN
THE SIMILAR DISEASE SPACE THE
MUTOSIS SPACE WE HAD PREVIOUS
EXPERIENCE SO TAKING THE
VOLUMETRIC LESION SHRINKAGE
COUPLED WITH ADDITIONAL
PHYSICIAN-REPORTED DATA WAS
EXTREMELY HELPFUL IN
SUPPLEMENTING THE IMAGE BASED
RESPONSE AND ASSESSMENT OF
EFFICACY GIVEN THE HETEROGENEITY
IN THE PHENOTYPE OF PATIENTS ONE
THIS PROSE AND THAT MAY BE
APPLICABLE TO OTHER LYMPHATIC
DISEASES AS WELL.
I'LL STOP THERE.
>> I HAVE ONE FOLLOW-ON
QUESTION, TO WHAT YOU WERE
TALKING ABOUT.
WITH LYMPHEDEMA WE HAVE AN
UNUSUAL SITUATION WHERE IT NOT A
RARE DISORDER BUT DON'T HAVE
GREAT OBJECTIVE METRICS
PARTICULARLY TO RELATE TO
THERAPEUTIC RESPONSE.
AND ONE OF THE BIG PROBLEMS IS
THAT HISTORICALLY ENSHRINED IN
THE LYMPHEDEMA LITERATURE IS
PHYSICAL MEASURES AND THE SIGNAL
TO NOISE RATIO THERE IS
PROBLEMAT EC IN TRACKING
THERAPEUTICS.
THAT WAS A BIG INTRODUCTION TO
ASK WOULD THE FDA BE OPEN TO NEW
METRICS EVEN IF THEY'RE NOT
HISTORICALLY ENSHRINED IN THE
EXISTING LITERATURE IF THEY'RE
DEVELOPED TO BE MORE SENSITIVE
TO THERAPEUTIC RESPONSE AND
TREATMENTS LIKE THAT.
>> UNDERSTANDING THE FDA BASED
ON ITS STATUTORY AND REGULATORY
RESPONSIBILITIES WE HAVE WE HAVE
TO GAUGE ON HOW IT MAY HELP THE
PATIENT FEEL FUNCTION OR SURVIVE
BETTER.
WHAT'S NOT BUILD IN TO THAT IS
THINGS THAT ARE PHARMACO DYNAMIC
MEASURES AND IMAGING
TECHNOLOGIES AND MEASUREMENTS OF
VOLUMES AND CHANGES OF VOLUMES
ARE NOT NECESSARILY DIRECTLY
CONNECTED TO PATIENT FEELING OR
FUNCTIONING OR SURVIVING BETTER.
GIVEN WE'RE LIKELY TALKING ABOUT
SYMPTOMATIC AND/OR FUNCTIONAL
BENEFITS HERE, THAT'S WHERE YOU
CAN USE THAT DATA TO SUPPLEMENT
A TOOL YOU BUILD AROUND THE
SYMPTOMATIC AND FUNCTIONAL
BENEFITS THROUGH A PRO OR ACRA.
WE DO LOOK APT -- AT THAT
EVIDENT AND SUPPORTED OR
CONFIRMATORY EVIDENCE THAT CAN
BE POWERFUL AS DR. SINGH HAD
SAID IN DETERMINING WHAT WOULD
LEAD TO SUBSTANTIAL EVIDENCE AND
EFFECTIVENESS AND DETERMINATION
TO FOLLOW ALONG, FDA HAS A
WEALTH OF GUIDANCE FOR
INDUSTRIES WE PUBLISH THAT ARE
PUBLICALLY AVAILABLE.
I RECOMMEND EVERYONE TAKES A
LOOK AT THOSE INCLUDING THE
SUBSTANTIAL EVIDENCE AND
EFFECTIVENESS FOR LOOKING AT ONE
ADEQUATE AND WELL CONTROLLED
STUDY WHICH IS PREDICATED ON
CONFIRMATORY EVIDENCE AND SOME
OF THAT DATA CAN SERVE AS
CONFIRMATORY EVIDENCE TO SUPPORT
OUR UNDERSTANDING OF THE
SYMPTOMATIC BENEFIT AND THERE'S
GUIDANCE ON CLINICAL OUTCOME
ASSESSMENT TOOLS AND SOME ARE
SPECIFIC TO THE TYPE OF
THERAPEUTIC OR TREATMENT OPTION
WHETHER THAT'S THROUGH THE
CENTER FOR DEVICES AND RADIO
LOGIC HEALTH FOR THE CENTER
THROUGH DRUG EVALUATION RESEARCH
OR BIOLOGICS AND RESEARCH WHICH
ARE MORE MARRIED IN TERMS OF HOW
THE GUIDANCES ARE BUILT.
>> DR. RICHARDSON, WE SEE YOUR
HAND UP.
>> JORDAN SAID ALMOST EVERYTHING
I WANTED TO SAY.
ONE THING WE HAVE TO REMEMBER IS
REGARDLESS OF WHETHER THE TEST
CHANGES OR NOT HOW DOES THE
PERSON FEEL WITH THE CONDITION
WHO IS BEING TREATED.
I THINK ONE OF THE REASONS WE
HAD SUCH A HARD TIME GETTING
TRACTION ON OUR HEALTH AND WELL
BEING AND PRIORITY WITH CANCER
SURVIVORS, THERE'S ALMOST ALL
SELF-REPORT.
SO WHEN PEOPLE TELL US THINGS WE
HAVE TO BELIEVE THEM AND AT
TIMES IF WE DON'T HAVE A QUOTE,
UNQUOTE, HARD OUTCOME SOMETIMES
WE DON'T BELIEVE PEOPLE AND WHAT
THEY'RE EXPERIENCING AND HOW
THEIR LIFE IS IMPACTED BY THE
CONDITION.
JUST TO REMEMBER THE PERSON'S
EXPERIENCES MAY BE MORE
IMPORTANT THAN OTHER THINGS
DISCUSSED.
>> THANK YOU FOR THE COMMENT.
WE APPRECIATE THAT.
OUR THIRD-PARTY COVERAGE
COMMITTEE HAD A NUMBER OF
QUESTIONS.
CAN I GET A REPRESENTATIVE FROM
THAT COMMITTEE TO ASK ONE OF WHY
YOU ARE QUESTIONS?
>> THOSE ARE QUESTIONS WE MUST
HAVE HAD.
>> TO OUR SCIENTIFIC ADVANCEMENT
COMMITTEE.
>> SO THANK YOU AND I JUST WANT
TO THANK EVERYONE FOR BEING HERE
AND CONTRIBUTING.
I'M GOING TO TRY TO SUMMARIZE
QUESTIONS AND STRUCK BY THE FACT
THE FEDERAL FUNDERS FOR
LYMPHATIC RESEARCH THE MAJOR
ONES ARE ALL HERE.
AS WE THINK ABOUT ONE OF OUR
GOALS AND CHARGES HERE IS TO
MAKE RECOMMENDATIONS AND WHAT
WE'VE SORT OF BEEN COALESCING
AROUND IS THE NEED FOR PERMANENT
RESEARCH INVESTMENT IN
LYMPHATICS.
AS I LOOK AT THE THREE
STRUCTURES FOR RESEARCH FUNDING
THAT ARE PRESENT HERE, WE CAN
SORT OF SEE PROS AND CONS.
WE'RE LOOKING FOR PERMANENCE.
WE'RE LOOK FOR MONEY DIRECTED
TOWARD A PROGRAM.
AND THE NIH IS NOT -- WE CAN'T
SAY NIH FUND LYMPHATIC RESEARCH
IT'S ALL GOING INTO POOLS AND
DOD HAS MORE OF THAT PROGRAM
ASPECT AND RELY ON CONGRESS TO
GIVE YOU THE BUDGET AND ARPA-H
IS TIME LIMITED.
I GUESS MY QUESTION IS AS WE SIT
WITH A VISION OF A PERMANENT
STRUCTURE FOR FUNDING LYMPHATIC
RESEARCH, WHAT ADVICE WOULD YOU
GIVE US, WHAT IDEAS DO YOU HAVE
FOR US?
DOES IT INCLUDE WORKING WITH
NONPROFITS?
WE'RE HOPING AND GRATEFUL FOR
ANY INPUT.
>> I THINK A REALLY IMPORTANT
THING IS DATA.
HOW ARE WE GOING TO ENSURE IT'S
A CENTRAL PLACE FOR EVIDENCE.
I WAS STRUCK WORKING ON THE
NATIONAL INDICATOR REPORT THAT
WE DON'T -- WE CONSENT REALLY
SAY WHAT THE PREVALENCE IS OF
SOMEONE WHO JUST UNDERWENT
CANCER THERAPY AND LIVING WITH
LYMPHEDEMA.
WE DON'T KNOW BECAUSE DATA ARE
IN SILOS.
WE HAVE A CHANCE AND AN
OPPORTUNITY NOW.
THERE'S NOW RESOURCES GOING INTO
LYMPHATICS AND HOW DOES IT LIVE
ON?
>> TO THAT POINT THOUGH BECAUSE
THINGS ARE SILOED, WHAT ADVICE
DO WE HAVE ABOUT
MULTI-INSTITUTIONAL
COLLABORATIONS BOTH FEDERAL
PARTNERS AND FLAUN -- NONPROFIT
TOEN THUR DATA IS GETTING OUT
THERE AND WHAT ARE LESSONS
LEARNED OF BIG BODIES BECAUSE A
LOT OF WORK IS BEING DONE BUT IN
DIFFERENT PLACES.
>> WE WANT TWO REACH OUT TO
PEOPLE LIKE THE NIH AND FDA AND
SEE WHAT ARE THE IMPORTANT
VARIABLES THAT SHOULD BE
COLLECTED THAT NIH MIGHT BE
COLLECTING RIGHT NOW WE CAN
STANDARDIZE AND ASK OUR TEAMS TO
ALSO COLLECT THE SAME DATA?
IF WE HAVE A COHORT, WE SHOULD
BE COLLECTING THE DATA THAT'S
STANDARDIZED AND REPRESENTATIVE
AS WELL.
REALLY FASCINATING.
I'VE HAD THE PRIVILEGE OF
WORKING WITH AN ACCESSIBILITY
EXPERT.
AS AN ACADEMIC I NEVER THOUGHT
ABOUT HOW WE GO OUT AND GET THE
PATIENTS THAT ARE PART OF OUR
COHORT.
WE COLLECT IT GENERALLY FROM OUR
CLINICS.
THAT'S A BIASSED SAMPLE.
SO WHAT WE'VE BEEN WORKING ON IS
BIG PICTURE AND SAMPLE FROM
REPRESENTATIVE POPULATIONS
WITHOUT PUTTING AN ONUS ON THE
ACADEMIC PERSON, FOR EXAMPLE,
BUT HOW DO WE DO IT BETTER?
BECAUSE THE TECHNOLOGY IF IT'S
BASED ON ONE CLINICAL COHORT IT
WON'T WORK ON SOMEONE ELSE IN
THE RURAL AREA.
WE'RE THINKING BIG LIKE THAT AND
THEN AGAIN BRINGING IN PATIENTS
AND EARLY INVESTIGATORS THE IDEA
IS WE'RE GOING TO BUILD A TRUST
AND WITH RURAL AREAS AND BRING
THEM IN AND I WAS TRYING TO
BRING IT ALL IN TOO.
>> WE CAN'T FORGET THE POWER OF
PATIENT ADVOCACY.
IT'S FRUSTRATING FOR ME WE'RE
COMPLETELY DEPENDENT ON ADVOCACY
GROUPS AND WHEN YOU TALK ABOUT
CONTINUITY OF RESEARCH I CAN'T
LOBBY FOR FUNDS.
WE'RE FEDERAL EMPLOYEES WE CAN'T
DO THAT BUT PATIENT ADVOCACY
HAVE THE LOUDEST VOICES.
I THINK WE ARE AT AN AGE WHERE
WE'RE STILL MISSING THE
OPPORTUNITY TO PARTNER WITH THEM
AND TO GET BETTER CONTINUITY OF
RESEARCH FROM FUNDING.
THEY'LL HAVE THE LARGEST WEIGHT
ON THIS.
>> KIM TO FOLLOW ON WHAT YOU
WERE TALKING ABOUT, I CONCUR ON
CENTRALIZATION OF DATA.
ONE OF THE PROBLEMS WE FACE HERE
IS A LACK OF ENUMERATE OF SOME
OF THE DATA WE MOST REQUIRE.
IT'S NOT SIMPLY POOLING WHAT
EVERYBODY HAS.
WE KNOW BIG DATA SETS DRIVE
EPIDEMIOLOGY AND MINEABLE FOR
INFORMATION BUT AMONG THE
DIFFERENT ORGANIZATIONS WHO HAS
WHAT'S BEING CAPTURED THAT'S NOT
CAPTURED IN REIMBURSEMENT DATA
SETS AND HOW DO WE MAKE THAT
HAPPEN TO FINALLY UNDERSTAND THE
MAGNITUDES OF THE PROBLEMS WE'RE
FACING.
>> A FOLLOW-UP QUESTION.
WE'RE EXCITED THE DOD HAS LOOKED
AMOUNT LYMPHATIC DISEASES.
ORIGINALLY TWO YEARS AGO THE
LYMPHATIC DISEASES WERE A PART
OF THE DOD PORTFOLIO AND RIGHT
NOW IT'S NOT THERE LONGER.
HOW DOES THE MECHANISM HAPPEN?
>> ALL THAT COMES TO THE PROBLEM
IS AS A RESULT OF THE ADVOCACY
GROUP.
WE WORKED CLOSELY WITH LEARN AND
THE TOPIC AREAS HOW THEY ENDED
UP FOLLOWING BEHIND PRN AND
THERE'S LITTLE CONGRESS ASKS US.
WE'RE ALWAYS HERE TO EXECUTE.
AND FY23 YOU WERE CORRECT WE HAD
LYMPHEDEMA AND LYMPHATIC DISEASE
WHICH WAS A HAUJ FEAT NOR
COMMUNITY BECAUSE IT'S NOT OFTEN
WE HAVE -- I'M NOT SAYING
THEY'RE OVERLAPPING BUT IT'S NOT
OFTEN WE GET IN A PROGRAM WITH
42 TOPIC AREAS AND NO DEDICATED
FUNDING FOR EACH YOU'D HAVE AN
OPPORTUNITY WITH ANY TWO.
AND THAT WAS HUGE.
THE FOLLOWING YEAR WE JUST ENDED
UP WITH LYMPHEDEMA, NO LYMPHATIC
DISEASE ANYMORE AND IN THE
INTEREST OF FULL DISCLOSURE, WE
HAVE A PROGRAM FROM THE SENATE.
WE HAVE THE SENATE LANGUAGE AND
REPORT THIS YEAR AND NONE OF
THEM WERE ADDED TO THE POTENTIAL
TOPIC AREAS IN FY25.
I KNOW THERE'S A HUGE EFFORT AND
ADVOCACY TO PUT THEM BACK IN THE
LANGUAGE.
I HOPE THAT WE GET TO THAT BUT
RIGHT NOW WE ARE BASICALLY
DEPENDENT AND WHY I SAY
PARTNERING WITH ADVOCACY GROUPS
AND THERE'S OTHER MEANS AND
HOWARD HUGHES FOR INSTANCE THEY
INVEST IN PEOPLE NOT PROJECTS.
THERE'S OTHER WAYS OF FUNDING WE
CAN MAKE SURE CONTINUITY AT
RESEARCH THAT WE AT DOD CAN'T.
>> IT'S CONGRESS?
>> IT'S CONGRESS.
CONGRESS HAS THE POWER OF PURSE.
IT'S ALWAYS CONGRESS.
BUT THE DECISION IS BASED ON HOW
MUCH LOBBYING GOES BEHIND IT I
WOULD SAY.
>> ONE OF THE THINGS WE ALWAYS
DISCUSSED IS PARTNERING WITH
FEDERAL AGENCIES WITH OUTSIDE
AGENCIES.
DOES THAT HAPPEN AT ALL THROUGH
DOD OR ARPA-H OR NIH?
IS THERE ANY MECHANISM
PRECEDENCE FOR CDC OR ANY OF
THOSE STAKEHOLDERS HERE WHERE
OUTSIDE AGENCIES ARE PARTNERING
WITH FEDERAL AGENCY TO DRIVE A
RESEARCH AGENDA?
>> SO I WILL SPEAK FOR THE DOD,
I SHOWED A SLIDE THAT HAD ALL
THE STRATEGIC GOALS WHEN YOU
TALK ABOUT OUTSIDE AGENCIES.
I WILL INCLUDE AND KIM CAN
ATTEST TO THAT INCLUDING OTHER
AGENCIES AN ADVOCACY GROUPS.
I HAVE ROUNDTABLE DISCUSSIONS
EVERY YEAR WITH ADVOCACY GROUPS
UNDER EACH PORTFOLIO ASKING WHAT
IS IT YOUR FIELD NEEDS OR
COMMUNITY NEEDS?
MAKING SURE WE'RE CAPTURING IN
OUR SOLICITATION TO ENSURE THE
NEEDS OF THE COMMUNITY ARE
CAPTURED IN THE SOLICITATION.
THAT ENSURES WE RECEIVE A LOT OF
PROPOSALS AND IN FY23 WE
RECEIVED 19 PROPOSALS IN
LYMPHATIC DISEASE AND LYMPHEDEMA
COMBINED.
I THINK THERE'S MORE OUTREACH WE
CAN DO.
WE PROVIDER WEBINARS TO THE
RESEARCHERS IN THE COMMUNITY TO
MAKE SURE THEY KNOW AND THOSE
WEBINARS ARE ORGANIZED THROUGH
ADVOCACY GROUPS.
THEY KNOW THERE'S A FUNDING
OPPORTUNITY AVAILABLE BUT STILL
ONLY RECEIVED 19 PROPOSALS.
WE ENDED UP FUNDING OVER FIVE SO
WE DID HAVE A VERY GOOD SUCCESS
RATE AND FUNDING IF YOU TAKE
INTO CONSIDERATION IT'S A
PROGRAM WITH 42 TOPIC AREAS.
>> I'LL ADD THAT WE'LL HEAR MORE
ABOUT THOSE PARTNERSHIPS IN THE
NEXT PANEL AS WELL.
>> WE HAVE TIME FOR ONE LAST
QUESTION.
>> CAN YOU COMMENT ON THE
APPROVAL PROCESS COMPARING
SYSTEMIC MEDICATIONS VERSUS
TOPICAL DRUGS OR EVEN DEVICES?
THERE'S DIFFERENT RULES FOR
SYSTEMIC VERSUS TOPICAL
MEDICATIONS AND CAN YOU COMMENT
ON THAT?
>> MANY TOPICAL PRODUCTS BOTH
SYSTEMIC AND TOPICAL PRODUCTS
WILL END UP IN THE SAME DIVISION
UNLESS THEY HAVE A SPECIFIC
DETERMINATE -- DERMATOLOGICS
AND THE SAME REGULATORY
STANDARDS WILL APPLY IDENTIFYING
WHAT YOUR TREATMENT INDICATION
THROUGH THE POPULATION IS AND
DEMONSTRATING IN DOSE AN
EFFICACIOUS RESPONSE AND IS
SAFE.
THINKING ABOUT SAFETY WITHOUT
SYSTEMIC EXPOSURE YOU MAY HAVE
LESS OF A RISK PROFILE THAN YOU
WOULD HAVE OTHERWISE.
YOU CAN UNDERSTAND A BASE NUMBER
OF SAFETY EVENTS WITH USE.
WITH THE DEVICE SIDE I CAN'T
SPEAK SPECIFICALLY TO THAT BUT
TYPICALLY CLINICAL TRIALS WITHIN
DEVICES ARE SMALLER AND OFTEN
TIMES THEY DON'T HAVE THE SAME
REQUIREMENTS FOR COMPARATOR
ARMS.
AGAIN, IN THE DRUG SPACE IT'S
GOING TO BE SIMILAR BETWEEN
TOPICAL AND SYSTEMIC AND ASK
CECELIA TO COMMENT IF SHE HAS A
DIFFERENT PERSPECTIVE.
>> YOU CAPTURED IT WELL, JORDAN.
FOR THE MEDICAL PRODUCT WE COME
BACK TO SUBSTANTIAL EFFORT AND
EFFECTIVENESS AND HOW IT CAN BE
DEMONSTRATED CAN VARY AND WHERE
THE DISCUSSION HELPFUL.
I THINK YOU CAN TREAT IT WELL.
THANKS.
>> THANK YOU FOR STICKING WITH
US AT LEAST 15 MINUTES PAST WHAT
WE PROMISED.
WE APPRECIATE IT.
I KNOW THERE ARE ADDITIONAL
QUESTIONS AROUND THE ROOM SO TO
OUR PANELISTS NOT ONLY WILL I
THANK YOU FOR BEING WITH US
TODAY BUT IN ADVANCE FOR
RESPONDING TO FOLLOW-UP
QUESTIONS WHICH I'M SURE YOU'LL
GET OVER E-MAIL.
AS WE MOVE TO OUR BREAK I'LL
PASS THE MIKE TO SELEN.
>> THANK YOU, EVERYONE AND
ESPECIALLY OUR STAKEHOLDERS.
THANK YOU FOR ME PESTERING YOU
TO COME TO THIS SESSION AND YOU
CAN SEE THIS IS A VERY IMPORTANT
DISCUSSION AND HOPE THIS WAS THE
INITIAL CONVERSATION AND WE'LL
SEE HOW THERE'S A LARGE NEED IN
THE LYMPHATIC RESEARCH AREA BUT
ALSO UNDER LIES THAT THOUGH THIS
IS A LYMPHATIC DISEASE WE'RE
TALKING ABOUT IT MAY BE
APPLICABLE TO MANY DISEASES THAT
PEOPLE ARE IN NEED FOR THEIR
WELL BEING.
I HOPE WE CAN CARRY THE COVERING
ON AND YOU WILL BE CONTACTED.
THANK YOU.
>> THANK YOU.
>> WITH THAT OUR OPEN SESSION
ENDS.
WE'LL HAVE A LUNCH BREAK
FOR EVERYONE IN THIS ENVIRONMENT
USE THE SEND FEEDBACK BUTTON AND
THE QUESTIONS WILL BE RECORDED
AND TAKEN INTO CONSIDERATION BY
THE COMMISSION AFTER THE
MEETING.
THIS VIDEO WILL BE ARCHIVED FOR
NIH COLLEAGUES FOR AN INFINITE
AMOUNT OF TIME AND WE'LL START
THE SECOND PANEL.
I'LL PASS THE MIKE TO OUR
CO-CHAIR DR. STAN ROCKSON.
>> THIS IS AN INCLUSIVE
ACCESSIBLE FORUM TO ADDRESS
CHALLENGE IN SCIENTIFIC AND
CLINICAL OPPORTUNITIES.
WE HOPE TO GATHER INPUT FROM A
WIDE AND DIVERSE GROUP OF
STAKEHOLDER TO IDENTIFY GAPS AND
CHALLENGES FOR ACCESSIBLE
LOGISTIC DISEASE DETECTION,
DIAGNOSIS AND PREVENTION FOR ALL
POPULATIONS AN INCLUSIVE FORUM.
AND REPORT OUT TO THE NATIONAL
HEART, LUNG AND BLOOD INSTITUTE
UNIVERSITY COUNCIL.
WE'RE COMPRISED AS A COMMISSION
SUBDIVIDED INTO COMMITTEES AND
SEVERAL OF OUR COMMISSION MANY
EMBERS SERVE ON MULTIPLE
COMMITTEES.
THESE ARE SCIENTIFIC
ADVANCEMENT, EDUCATIONAL
IMPERATIVES, MEASURABLE RESULTS,
THIRD PARTY AND COMMUNITY GROWTH
AND YOU'LL SEE ON THE SLIDES
INDICATED ON THE CHAIRS OF THE
PERSPECTIVE COMMITTEES.
WITH THAT IN MIND I'D LIKE TO
MOVE TO PANEL B AND EXPLORING
DIFFERENT MODELS TO ADVANCE
LYMPHATIC RESEARCH DISEASES AND
THIRST IS LAURA LYMAN RODRIGUEZ
FOR STRATEGIC PLANNING AND
OUTREACH OF THE OF THE
PATIENT-CENTERED OUTCOMES
RESEARCH INSTITUTE.
>> CAN EVERYONE HEAR ME OKAY?
WHAT IS TO OUR NAME IS PATIENT
CENTEREDNESS.
I WILL PROVIDE REMARKS TO
PROVIDE THAT HIGH LEVEL
BACKGROUND INFORMATION FOR THOSE
WHO MAY NOT BE FAMILIAR WITH P
CORRI BUT TRY TO KEEP IT LIFE --
PCORI AND DURING THE Q&A I CAN
PROVIDER MORE INFORMATION.
-- PROVIDE MORE INFORMATION.
TO START WITH THE BASICS LOOKING
AT OUR MISSION AND THAT IS
REALLY GROUNDED IN THE CONCEPT
OF PROVIDING INFORMATION TO
PATIENTS, CAREGIVERS, PROVIDERS,
ALL SECTORS OF THE HEATH
RESEARCH AND PARTICULARLY HEALTH
CARE COMMUNITY TO INFORM
EVIDENCE-BASED DECISION MAKING
ABOUT HEALTH TOE IMPROVE HEALTH
OUTCOMES.
THAT'S THE GROUND IING FOUNDATI
HOW PCORI'S WORK BEEN
ESTABLISHED AND ORGANIZED AND
THE GOAL OF PATIENT CENTEREDNESS
IMPROVING HEALTH OUTCOMES
THROUGH THAT LEVER OF INFORMED
DECISION MAKING BY ALL PARTIES.
IN TERMS OF BEING FULLY FUNDED
BY THE FEDERAL GOVERNMENT BUT
NOT A FEDERAL AGENCY, SO WE ARE
A NON-PROFIT ORGANIZATION
ESTABLISHED UNDER THE AFFORDABLE
CARE ACT IN 2010 AND FUNDING WAS
REAUTHORIZED THROUGH 2029 IN
2019.
THIS COMES FROM APPROPRIATIONS
AS WELL AS THROUGH FEES TO THE
PAIR COMMUNITY THAT COME IN AND
ALSO FUNDING FOR THE
PATIENT-CENTERED TRUST OUTCOME
AND SUPPORT PCORI AND THE HEALTH
CARE FOR EIGHTH AND EQUALITY AND
THE DEPARTMENT OF HEALTH AND
HUMAN SERVICES PARTICULARLY
AROUND DATA INFRASTRUCTURE.
SO WITHIN THE UNLYING STATUTE
ARE SPECIFIC PURPOSES FOR THE
THREE ENTITIES.
WITH PCORI FOCUSSED ON THE
PATIENT-CENTERED COMPARATIVE
CLINICAL EFFECTIVENESS RESEARCH
AND WE'LL TALK ABOUT THAT ON THE
NEXT SLIDE.
AND SINCE WE BEGAN RESEARCH
AWARDS WE FUNDED OVER 2400
AWOORDZ AS OF -- AWARDS AT THE
END OF AUGUST FOR RESEARCH AND
RESEARCH-RELATED ACTIVITIES.
I'LL TALK ABOUT THAT AS WELL.
CLINICAL EFFECTIVENESS RESEARCH
TO LEVEL SET FOR EVERYONE, THAT
IS IN TERMS OF THE YOU RESEARCH
SPECTRUM BETWEEN BASIC RESEARCH
AND CLINICAL PRACTICE.
IT'S DISTAL TO RESEARCH COMING
FROM NIH FOR A LONG TIME SEEM TO
BE AT THE FAR END OF THINGS.
WE DO LOOK SPECIFICALLY AT
COMPARING TWO DIFFERENT
TREATMENTS OR INTERVENTIONS AND
LOOKING AT WHAT IS THE MOST
EFFECTIVE IN A GIVEN SCENARIO OR
DIFFERENT GROUPS WITHIN THE
STUDY POPULATION AND FOCUSSING
ON THE DIFFERENT POPULATIONS
UNDERSTANDING HETEROGENEITY OF
EFFECT AND UNDERSTANDING WITH
THE GOAL TO THINK ABOUT WHAT
WORKS BEST FOR EACH INDIVIDUAL
AND THEIR PARTICULAR
CIRCUMSTANCES WHICH AGAIN GOES
BACK TO OUR PATIENT CENTEREDNESS
LESS FOR EVERYTHING WE DO.
WE DO NOT MAKE POLICY
RECOMMENDATIONS OR MAKE
RECOMMENDATIONS WITH REGARD TO
CLINICAL GUIDELINES AND THAT'S
IMPORTANT TO UNDERSTAND.
WE VERY MUCH INTEND FOR THE
EVIDENCE THAT IS GENERATED
THROUGH PCORI FUNDED RESEARCH TO
BE INFORMATIVE TO THE DECISION
MAKERS MAKING DECISIONS IN THE
POLICY REALM.
AND LIKEWISE WE'RE FOCUSED AND
ENCOURAGED EN OUR RESEARCH FOR
ADVOCATORS TO CONSIDER THE
PATIENT BURDENS OR OTHER
OUTCOMES RELEVANT TO THE STUDY
AND SO LOOK AT COST
EFFECTIVENESS RESEARCH ITSELF.
AND A FULL PORTFOLIO AND SUITE
OF ACTIVITIES BECAUSE ONE OF THE
OTHER THINGS THAT MAKES PCORI
DIFFERENT FROM OTHER RESEARCH
FUNDERS IS LOOKING BEYOND THE
RESEARCH ITSELF AND FOCUS ON
DISSEMINATION AND IMPLEMENTATION
TO CARRY FORWARD THAT PATIENT
CENTERED APPROACH TO MAKE SURE
THAT INDIVIDUALS WHETHER
PATIENTS OR PROVIDERS, HEALTH
SYSTEMS, ETCETERA HAVE THE
INFORMATION THEY NEED TO INFORM
DECISIONS THAT THEY'RE MAKING
ABOUT HEALTH.
THERE'S AN EXTENSIVE STRATEGIC
PLANNING ACTIVITY INVOLVING
CANVASSING THE DIFFERENT SECTORS
WITHIN PCORI'S COMMUNITY AND
PATIENT AND ADVOCACY COMMUNITIES
AND SCIENTIFIC AND POLICY
COMMUNITY, ETCETERA, TO
UNDERSTAND WHERE WERE THE
GREATEST OPPORTUNITIES FOR
COMPARATIVE CLINICAL
EFFECTIVENESS RESEARCH TO FOCUS
AGAIN ON PATIENT CENTERED
HEALTH.
IT'S NOT A DISEASE LENS WE BRING
TO THE WORK WE DO BUT LOOKING AT
THE WHOLE PERSON HEALTH AND AT
NATIONAL PRIORITY FOR HEALTH.
THE HIGHEST LEVEL OBJECTIVES WE
HAVE AND THERE'S FIVE GOALS
HERE.
I WON'T TALK IN DEPTH ABOUT THEM
TODAY BUT THEY ARE ALL VERY
BROAD AND LONG TERM ASPIRATIONAL
OBJECTIVES WE HAVE AND WHAT'S
IMPORTANT ABOUT THEM TOO IS
THEY'RE MUTUALLY REINFORCING.
YOU CAN'T TALK ABOUT ONE WITHOUT
ASPECTS OF THE OTHER.
WE APPROACH THESE IN COLLECTIVE
WAY VERSUS A GOAL BY GOAL
FACTOR.
PART OF HOW WE DO THAT AND I
WILL SHARE MORE DETAILS ON THIS
HAVE TO DO WITH THE WHOLISTIC
APPROACH WE BRING TO SHAPING THE
PORTFOLIO.
THE OTHER CORE ANCHOR,ING, IF
YOU WILL, THAT GUIDES PCORI AND
PARTICULARLY ITS RESEARCH
ACTIVITIES THE RESEARCH AGENDA
AGAIN COMING THROUGH THE
PERSON-CENTERED HEALTH LENS AND
THINKING HOW WILL ANY OF THE
RESEARCH AFFECT PATIENT SLIDES,
THE CHOICES PATIENTS AND
FAMILIES ARE FACING AS THEY
NAVIGATE ANY HEALTH JOURNEY AND
OVER THE LIFE SPAN.
THIS IS A FRAMEWORK THAT GUIDES
THE RESEARCH THAT WE FUND AND
NOT AN EXTENSIVE LIST OF
PROJECTS WE FUND.
I WANT TO TALK ABOUT SOME OF HOW
WE GET TO THE TOPICS WE FUND
THROUGH OUR FUNDING ANNOUNCE.
S TO TALK ABOUT HOLISTIC
APPROACH.
THIS IS HOW WE EMBODY THE
MISSION STATEMENT THAT I TALKED
ABOUT INTO THE WORK WE DO.
AND THE FOUR PEDALS OF THE WHEEL
ARE FOCUSSED ON ALL THE NATIONAL
PRIORITIES FOR HEALTH.
ALL FIVE OVERARCHING OBJECTIVES
AND NOT ANY ONE IN ISOLATION.
I'LL START WITH ENGAGEMENT
BECAUSE IT'S THE OIL, IF YOU
WILL, THAT GOES THROUGH THE
WHOLE MACHINE OF OUR ACTIVITIES
TO MAKE ALL OF IT WORK.
THIS IS MAKING SURE PCORI IT'S
ENGAGED IN THE EVERY STEP OF THE
RESEARCH WE FUND INTEGRATING
PATIENT CENTEREDNESS IN THE WORK
WE DO.
THIS HAPPENS FROM YOU'VE HEARD
SOME OF HOW IT HAPPENED IN HOW
WE AS AN ORGANIZATION THINK AND
PLAN WHAT THE AGENDA WILL BE FOR
THE INSTITUTE BUT ALSO
THROUGHOUT THE RESEARCH
PROCESSES.
EXPECTING PATIENTS ARE INVOLVED
IN THINKING ABOUT WHAT STUDY
PROPOSALS CONFIRM AND THERE'S
MEANINGFUL PARTNERSHIPS WITH
PATIENTS AND COMMUNITIES WITHIN
EVERY STUDY AND THEY'RE ASSESSED
ON THOSE PLANS DURING MERIT
REVIEW AND THAT INCLUDES PATIENT
AND OTHER MERIT REVIEWERS
LOOKING AT THE PROJECTS AND
RATING THE PROJECTS AS THEY COME
THROUGH.
THIS GOES ON INTO STUDY CONDUCT
AFTER FUNDING DECISIONS ARE MADE
AND THROUGH OUR PEER-REVIEW
PROCESS, PCORI LOOKING PAST
STANDARD PUBLICATIONS AND HAVE A
PEER REVIEW PROCESS EVERY STUDY
IS FUNDED THROUGH AND LEADS TO
FINAL RESEARCH REPORTS OF EVERY
STUDY PUBLISHED ON OUR WEBSITE
TO MAKE SURE THE FINDINGS,
POSITIVE OR NEGATIVE WHETHER
THEY END UP IN ANOTHER
PUBLICATION OR NOT HAVE
AVAILABLE TO THE PUBLIC THROUGH
THE WEBSITE.
GOES ON TO THINKING OF
DISSEMINATION AND POLITICIAN
ANOTHER OF THE PEDALS AND NOT
ONLY FOCUS ON THE WAYS WE CAN
DISSEMINATE FROM THE STUDIES AND
FUND AWARDS TO SUPPORT
DISSEMINATION OF FINDINGS IN THE
FORM OF ENGAGEMENT AWARDS TO
COMMUNITIES FOR THE STUDY
POPULATIONS THAT WERE INVOLVED
IN DIFFERENT STUDIES, TO
DISSEMINATE AND MAKE FOLKS AWARE
AND MOVE TO IMPLEMENTATION
ACTIVITY TO TRY TO IMPLEMENT THE
FINDINGS IN DIFFERENT SCENARIOS
AND ONE OF THE ACTIVITIES WE'RE
PARTICULARLY EXCITED ABOUT RIGHT
NOW BECAUSE IT'S NEW AND IT'S
INNOVATIVE IN HOW IT THINKS OF
SCALING IMPLEMENTATION IS OUR
HEALTH SYSTEM INITIATIVE AND
WHERE WE HAVE 42 CENTERS AROUND
THE COUNTRY THAT WE'LL BE ABLE
TO TAKE SPECIFIC FINDINGS THAT
SHOW PROMISE FOR IMPLEMENTATION
BASED ON EARLIER WORK TO SCALE
IMPLEMENTATION ACROSS MANY
DIFFERENT TYPES OF HEALTH CARE
SETTINGS, MANY HOI TO SCALE AND
SCOPE GOING FORWARD SO A
POWERFUL WAY TO MOVE
PCORI-FUNDING AND ENABLE CER TO
GO FORWARD IN A PATIENT-CENTERED
WAY INCLUDING WORKFORCE AND
RESEARCH INFRASTRUCTURE A
FLAGSHIP COMPONENT IS THE
PATIENT CENTER OUTCOMES RESEARCH
NETWORK.
AGAIN, A NATIONAL NETWORK OF
NETWORKS IN THIS CASE THAT
INCLUDES SITES SPANNING
DIFFERENT TYPES OF CLINICAL CARE
SETTINGS.
SPANNING DIFFERENT POPULATIONS
REPRESENTATIVE OF THE U.S. TO BE
ABLE TO HAVE A SYSTEM THAT
BRINGS IN LONGITUDE INTO THE
INFORMATION FROM ELECTRONIC
HEALTH RECORDS ACROSS THE
COUNTRY ACCESSIBLE TO BE STUDIED
BY ANYONE IN THE COUNTRY.
SO THEY CAN COME IN AND MAKE
REQUESTS TO SEE INFORMATION
ABOUT THE DATA THAT ARE WITHIN
PR C -- PCORNA AND THE PATIENTS
ARE CONSENTED FOR ADDITIONAL
STUDIES AT THE SITES SO IT'S
POSSIBLE AS INVESTIGATORS MAYBE
COME FORWARD THROUGH THE FRONT
DOOR TO LEARN ABOUT PATIENT
POPULATIONS AND WHAT DATA MAY BE
AVAILABLE TO INITIATE NEW
STUDIES AND COLLABORATIONS WITH
THOSE ADVOCATORS.
-- INVESTIGATORS.
IT'S A UNIQUE RESOURCE TRYING TO
PROMOTE THAT CER WITH THE
PATIENT-CENTERED CORE THAT IS
NATIONAL IN SCOPE AND SCALE.
IT'S ALSO AT THE READY TO BE
USED.
BEFORE GOING FORWARD THINKING
HOW TO MOVE FROM THE HIGH-LEVEL
OBJECTIVES UP HAVING INPUT FROM
THE PUBLIC AND SPECIFIC
COMMUNITIES AND THINKING ABOUT
THE RESEARCH TOPICS THROUGH
WHICH WE'LL SEEK TO ADVANCE THE
GOALS.
IT'S STILL DEPENDENT ON AN
ONGOING STREAM OF ENGAGEMENT AND
INTERACTIONS AND DIALOGUE WITH
OUR COMMUNITIES.
BRINGING IN IDEAS UP AN ONGOING
WAY, CONTINUOUS WAY FROM
PATIENTS AND STAKEHOLDERS AND
CONSIDERING WHAT FUNDING WE HAVE
ALREADY PUT IN PLAY WHETHER WE
HAVE NEW QUESTIONS OR MAKING
SURE WE'RE SPENDING THE PUBLIC
RESOURCES WISELY.
ALL OVERSEEING THE BOARD OF
GOVERNS AND TO NOTE WE HAVE AN
OVERSIGHT BODY OF INDIVIDUALS
MAKING MANY OF THE PCORI
STAKEHOLDERS AND BRINGING THEM
FORWARD TO BEAR AND THIS HELPS
US STAY IN TOUCH IN A REGULAR
AND ONGOING WAY WHERE THE NEEDS
ARE FOR CER AND IMPORTANT
QUESTIONS FACING PATIENTS AGAIN
IN THE WAY IT AFFECTS THEIR
LIVES AND NOT NECESSARILY NOT
ALWAYS JUST ABOUT A PARTICULAR
DISEASE BUT WHAT SCREENING
PROGRAMS ARE MOST EFFECTIVE AND
WHAT SYSTEM OF INTERVENTIONS
MAKE THE DIFFERENCE FOR WHOM AND
THINKING HOW WE DO THAT GOING
FORWARD.
THIS IS HOW WE SAY IN TERMS OF
WHERE PCORI FOCUSES ITS FUNDING
AND OUR APPROACH IS ALWAYS
HAVING AN OPEN CALL FOR IDEAS
READY TO GO FORWARD THROUGH
MENGFUL PARTNERSHIPS WITH
PATIENTS AND RESEARCHERS
ADDRESSING NEEDS AND THROUGH
FUNDING CYCLES WE HAVE OPEN CALL
FOR STUDIES AND LARGER
INITIATIVE TO COME FORWARD AND
BE REVIEWED THROUGH THE MERIT
REVIEW PROCESS UP ADDITION TO
SPECIFIC MORE FOCUSSED FUNDING
OPPORTUNITIES AROUND PARTICU%A
TOPICS AND WE'LL TALK ABOUT THEM
MORE AND CALL THEM THE
COLLECTION OF HIGH LEVEL TOPIC
THEMES THE RESEARCH PROJECT
AGENDA.
AND THAT'S MEANT TO BE A DYNAMIC
TOOL RELYING ON THE RESEARCH
AGENDA TO GUIDE THE INDIVIDUAL
FUNDING DECISIONS.
AND THEY'RE HIGH LEVEL AND HAVE
DIFFERENT CHARACTERISTICS IN
TERMS OF SPECIFICITY OF THE
HEALTH CONDITION.
ALSO IMPORTANT TO NOTE JUST
BECAUSE WE HAVE TOPIC THEMES
FOCUSSED ON HEALTH CONDITIONS WE
STILL CONSIDER IN EVERY
OPPORTUNITY THE CHARACTERISTICS
AND TRAITS IMPORTANT TO
PATIENT'S LIVES AND HOW WE LIVE.
AND THINKING ABOUT HOW DOES IT
AFFECT US AND HOW WE WORK, LIVE
AND PLAY GOING FORWARD.
AND WE UPDATED THE SET OF TOPIC
THEMES TO INCLUDE THE THREE IN
YELLOW.
WE EXPECT THIS TO BE A WAY TO
REMAIN RESPONSIVE TO WHAT THE
GREATEST AND HIGHEST PRIORITY
NEEDS ARE ACROSS THE COMMUNITY
FOR CER EVIDENCE TO COME
FORWARD.
I'LL TRANSITION TO TALK ABOUT
HOW THE PATIENT-CENTERED
APPROACH CAN AFFECT THE WORK WE
DO THROUGHOUT THE LIFE CYCLE.
THIS IS THE STUDY FOCUSSED ON
COMPARED ANTIBIOTIC USE VERSUS
SURGERY FOR PATIENTS WITH
UNCOMPLICATED AP PPEN PEND SIG
IS -- APPENDICITIS AND IT
ENGAGED WITH THREE PATIENT
GROUPS AT VARIOUS LEVEL
INCLUDING THE PROVIDER GROUPS
AND NATIONAL ADVISORY BOARD WITH
PROFESSIONAL SOCIETIES AND
POLICY MAKERS AND OTHERS AS WELL
AS THE PATIENT ADVISORY BOARD.
THINKING ABOUT THOSE IN
PARTNERSHIPS WITH VARIOUS LEVEL
AND CONSIDERING AS THE STUDY IS
CONCEIVED WHAT THE NEEDS WILL BE
AT THE END FOR DECISION MAKING
PROCESSES.
MY PURPOSE IN SHARING IS TO NOTE
TAKING TOGETHER CAN PROVIDE
USEFUL INFORMATION FOR PATIENTS
AND CAREGIVERS OF WHICH
TREATMENT HAVE TO FOLLOW.
THESE FINDINGS ALSO WERE
FORTUNATELY AVAILABLE AND TAKEN
UP DURING THE PANDEMIC AND
INCORPORATED INTO CLINICAL
GUIDELINES AS SOMETHING FOR
PROVIDERS TO CONSIDER WHERE IF
WE THINK THAT THE SHORT TERM
THERE WAS DEFINITELY STRONG
INCENTIVE WHY ANTIBIOTICS COULD
BE EQUAL TO SURGERY OVER TIME
BUT DURING THE PANDEMIC WEREN'T
BEING ADMITTED TO THE HOSPITAL
CARRIED A DIFFERENT RISK FACTOR
THAN PERHAPS NOW OR OTHER TIMES
IT'S UCHLS TO MAKE ASSESSMENT
THE BEST INTEREST OF THE
PATIENT.
THIS WAS TAKEN UP TO CLINICAL
GUIDELINES FOR SURGEONS AT THE
TIME TO BE CONSIDERED AND THAT'S
AN IMPORTANT MEASURE IN TERMS OF
THE UPTAKE HAPPENING AND IS THE
FUNDING PCORI IS PUTTING INTO
PLAY MAKING A DIFFERENCE FOR
PATIENTS IS THERE WAS AN
EVERYDAY UPDATE WE DISSEMINATED
TO DO WHAT WE COULD DO AND REACH
THE AUDIENCES WE COULD.
WE ALSO ARE WORKING ON
IMPLEMENTATION PROGRAMS TO TEST
IMPLEMENTATION AND LEARN HOW TO
GET THE INFORMATION OUT AND HAVE
IT AFFECT PATIENTS' LIVES AND
DAY TO DAY BASIS.
THERE'S AN EXAMPLE OF THE
DIFFERENT TOUCHES PCORI CAN HAVE
IN FUNDING THE RESEARCH THAT CAN
MAKE A DIFFERENCE AND INFORM
CHOICES THROUGHOUT THE CONDUCT
OF THE RESEARCH.
THERE'S ONE LAST FACET TALKING
ABOUT WHAT IS FUNDAMENTAL TO HOW
PCORI OPERATES AND KEEPS US TRUE
TO THE PATIENT-CENTERED APPROACH
AND LENS IS PCORI ACTING AS A
LEARNING ORGANIZATION.
I WON'T WALK THROUGH THE
COMPLICATED GRAPHIC AND TO
DEMONSTRATE ON THE LEFT SIDE WE
SEE THE LARGE HIGH-LEVEL OVER
ARCHING AND MUTUALLY REINFORCING
GOALS FOR THE NATIONAL
PRIORITIES OF HEALTH.
ON THE FAR RIGHT THE
ASPIRATIONAL IMPACT WE HOPE TO
HAVE IN TERMS OF IMPROVING
HEALTH AND MAKE SURE THERE'S
EQUITABLE HEALTH CARE IN PLACE.
WHAT'S AT THE CORE OF PCORI THE
CONSTANT AND ONGOING MONITORING
OF PROGRESS AND MEASURE SUCCESS
IN TERMS OF GETTING NFLS OUT
THAT'S FUL IN A TRUSTED CAPACITY
FOR PATIENTS TO USE.
AGAIN WE LOOK THROUGH THE
DIFFERENT STAGES OF THE RINGS
LOOKING TO SEE THE EVIDENCE
BEING PRODUCED THROUGH FUNDING
MAKING A DIFFERENCE TO PATIENTS
AND PROVIDERS.
ARE WE HELPING TO CREATE A
CULTURE WITH CAPACITY FOR
PATIENT-CENTERED CER AND IS IT
USED IN PRACTICE AND ARE WE ABLE
TO TRANSFORM THIS CULTURE AND
PATIENT CENTEREDNESS BEYOND THE
WORK WE FUND.
WE'RE A SMALL FUNDER SO WE HAVE
BIGGER DREAMS IN HOW TO HAVE AN
IMPACT.
THE LAST SLIDE LOOKING FOR THE
SIGNALS OF IMPACT AND FROM THE
RESULTS PERSPECTIVE WHAT WE CAN
DO TO MAKE SURE RESULTS ARE
DISSEMINATED IN THE FAR LEFT
MOVING TO RESULTS AND FINDINGS
TAKEN INTO PRACTICE AND HOW ARE
THEY HAVING AN IMPACT ON
PATIENTS AND HEALTH CARE.
THIS IS A LONG TERM PERSPECTIVE
AND THINGS WE DON'T HAVE ANSWERS
TO WE CAN TRACT.
THIS TAKES TIME.
SO THIS IS SOMETHING AS PCORI IS
NOW ENTERING ITS SECOND DECADE
WE'RE STARTING TO LOOK AT MORE
AND MORE CLEARLY BUT IT'S GOING
TO BE AN ONGOING PROCESS AND
THERE'S SO MUCH DATA YET TO COME
AND LEARN AND FEEDBACK INTO HOW
WE ARE DOING OUR DAY TO DAY
WORK.
I'LL STOP THERE.
>> THANK YOU, DR. RODRIGUEZ.
WE'LL HAVE QUESTION AND ANSWERS
AFTER THE PANEL PRESENTATIONS.
NEXT IS OUR SPEAKER.
>> I OVERSEE THE PARTNERSHIP
PROGRAMS WHEN I'LL TALK ABOUT
TODAY.
FOR THOSE WHO ARE NOT FAMILIAR
WITH THE FOUNDATION, WE'RE
CHARTERED BY CONGRESS IN 1996 TO
SUPPORT THE MISSION OF THE NIH.
WE HAVE THE UNIQUE ABILITY TO
TRANSFER FUNDING FROM PRIVATE
ENTITIES TO THE NIH AND VICE
VERSA AND WE USE THAT
FOUNDATIONAL WORK TO BUILD
PUBLIC-PRIVATE PARTNERSHIPS AND
FOCUS LARGELY IN THE
PRE-COMPETITIVE SPACE FOCUSSING
ON TRANSLATIONAL SCIENCE EARLIER
IN THE PROCESS.
WE WORK WITH MULTIPLE
STAKEHOLDERS, NIH OUR MOST
IMPORTANT PARTNER AND OTHER
GOVERNMENT AGENCIES AND FDA IN
PARTICULAR IS INVOLVED IN MANY
OF OUR PROGRAMS.
WE WORK WITH LIFE SCIENCE
COMPANIES AND ACADEMIA AND I
WOULD SAY MOST IMPORTANTLY WORK
WITH FOUNDATIONS AND PATIENT
ORGANIZATIONS.
MY FORMER LIFE FOR ALMOST A
DECADE I WAS THE CHIEF
INNOVATION OFFICER AT GENETIC
ALLIANCE WORKING ON GENETIC AND
RARE DISEASES AND SO WHEN I
JOINED THE FOUNDATION I WAS VERY
EXCITED ABOUT THE WORK THAT WE
DO TO MOVE THE NEEDLE IN THE
TRANSLATIONAL SCIENCE SPACE.
THIS IS A SPACE THAT PEOPLE
REACH INTO AND DABBLE INTO BUT
NO ONE TRULY OWNS AND WHAT WE
WORK ON IN THE SPACE- .
WE WENT EVERYONE AT THE TABLE
THROUGH TO IMPLEMENTATION AND
DISSEMINATION OF RESULTS.
THAT IS CORE TO WHAT WE DO.
WE HAVE RAISED A LITTLE OVER
$1.5 BILLION FROM PRIVATE
ENTITIES.
OBVIOUSLY THAT NUMBER IS LARGER
WHEN YOU CONSIDER THE PUBLIC
INVESTMENT THAT IS MADE ON
PROGRAMS THAT WE HELP
FACILITATE.
WE HAVE OVER 122 ACTIVE PROGRAMS
RANGING FROM $2 MILLION TO $220
MILLION OR LARGER IF YOU LOOK AT
SOME COVID WORK AND 90 CENTS OF
EVERY DOLLAR WE RAISE GOES
DIRECTLY TO THE PROGRAMS.
OUR WORK IS PRIMARILY IN THREE
AREAS.
AS MENTIONED IN THE
TRANSLATIONAL SPACE, PREVENTION,
THERAPIES AND DIAGNOSTICS AND
POTENTIAL CURES.
WE ALSO DO QUITE A BIT OF WORK
IN THE POPULATION HEALTH AND
GLOBAL HEALTH SPACE AND THEN WE
DO WORK TO CELEBRATE AND TRAIN
THE NEXT GENERATION OF
SCIENTISTS AND I'LL TOUCH ON
THOSE LATTER TWO BRIEFLY BUT I'M
GOING TO PRIMARILY FOCUS ON THE
FIRST TODAY.
IT'S CHALLENGING TO DISTILL INTO
THIS PRESENTATION WHILE GIVING A
SENSE HOW TO USE OUR MODEL.
WE'RE ENGAGED WITH OUR MODELS.
SO THE FIRST EXAMPLE IS A
PROGRAM THAT I OVERSEE THE
ACCELERATING MEDICINE
PARTNERSHIP AND WE JUST
CELEBRATED THE 10-YEAR AT THE
BEGINNING OF THIS YEAR.
WE FUNDED AND LAUNCHED 12
PROGRAMS RANGING FROM 50 MILLION
AND THE FUNDAMENTAL ASPECT IS
THEY HAVE INTEREST IN INVESTMENT
FROM ONE OR MULTIPLE INSTITUTE
OR CENTERS WITHIN THE NIH
MATCHED BY INVESTMENT AND
FUNDING FROM MULTIPLE COMPANIES
ACROSS THE SECTOR AS WELL AS
PATIENT ORGANIZATIONS.
AND THAT PARTNERSHIP IS CORE TO
THE MODEL.
EACH AIM DIFFERENT.
THERE'S QUITE A RANGE BUT FOR
THE MOST PART THEY LARGELY FALL
INTO TARGET SPACE, BIOMARKER
DEVELOPMENT SPACE, UNDERSTANDING
THE MOLECULAR UNDER PINNINGS OF
DISEASE AND THERE'S SOME
SKIPPINGSES.
THE SECOND PROJECT IS TO GIVE A
SENSE OF THE RANGE.
THE AMP BESPOKE GENE THERAPY
CONSORTIUM, FOR EXAMPLE, IS
FOCUSSED LATER IN THE
TRANSLATIONAL PIPELINE
ESPECIALLY FOCUSSED ON GENE
THERAPY FORERARE DISEASES THAT
MIGHT NOT HAVE -- FOR RARE
DISEASES AND LOOKING AT
STREAMLINING THE PATHWAY FOR
GENE THERAPY AND BY FIRST
RUNNING FIRST IN HUMAN TRIALS
FOR DIFFERENT DISEASE AREAS AND
35 PARTNERS AND INSTITUTES AND
COMPANIES AND PATIENTS.
IT'S QUITE A DIVERSE RANGE OF
PARTIES AND ALL THOSE
STAKEHOLDERS ARE ACTIVE
PARTICIPANT PANTS IN THE WORK
GROUP THEY'RE IN THE ADVISING
THEY'RE PARTICIPATING IN THE
DEVELOPMENT OF THE REGULATORY
FRAMEWORK.
I'LL TALK ABOUT AN EXAMPLE OF
THE MORE CLASSIC THE THIRD IS
THE BIOMARKER CONSORTIUM.
THEY FOCUSSED ON MORE DEFINED
CONTEXT AND USE, IDENTIFICATION
AND VALIDATION OF FIVE TYPES OF
BIOMARKERS.
WE HAVE FOUR FOUR THERAPEUTIC
AREAS WITHIN THE FOUNDATION AND
CANCER NEUROLOGY, IMPLEMENTATION
AND IMMUNITY AND METABOLICS WE
HAVE WORKING GROUPS IN RARE
DISEASE, WOMEN'S HEALTH.
AND HOW PROJECTS COME TO US AND
START WITH PARTICIPATING IN
INSTITUTE OR CENTER AND THE
BIOMARKER CONSORTIUM WE HAVE
STEERING COMMITTEES.
FOR EXAMPLE INFLAMMATION AND
IMMUNITY, WHICH I OVERSEE, EVERY
OTHER YEAR WE INVITE PEOPLE TO
BRING PITCHES AND INDUSTRY
STAKEHOLDERS AND ACADEMIC AND
GOVERNMENT STAKEHOLDERS AND LIVE
DESIGN THOSE PROGRAMS AT ROUND
TABLES AND FEASIBILITY AND
INTEREST AND COME UP WITH
PROJECTS THAT ALIGN THE INTEREST
AND INVESTMENT FROM THE PUBLIC
AND PRIVATE SECTOR TO BRING
TANGIBLE PROJECTS TO BE
IMPLEMENTED AND CHANGE CLINICAL
PRACTICE.
AND THEY REACH $3 TO $10 MILLION
IN SIZE.
OUR FDA AND NIH COLLEAGUES ARE
VERY ACTIVE EARLY IN THE WORKING
GROUPS AND STEERING COMMITTEE
FOR THE PROGRAMS AS WELL AND
LARGELY FUNDED BY PRIVATE SECTOR
OR PATIENTS.
AND WE HAVE PROGRAMS IN
OSTEOARTHRITIS AND FUNDING FOR A
PROGRAM IN SJOGREN'S RIGHT NOW
AND HAVE KIDNEY SAFETY AS WELL
AS AND BUILDING PROGRAMS IN
LUPUS AND SCLERODERMA AND A
NUMBER OF THINGS.
IN THE LEAD UP TO THAT MEETING
IT'S NOT LIKE AN RFP.
AND ONE STEP IS TO REACH OUT TO
STAKEHOLDER GROUPS WITHIN OUR
COMMUNITY AND WHO ARE YOU
FUNDING THAT WOULD BE
APPROPRIATE FOR PUBLIC-PRIVATE
PARTNERSHIP AND ACTIVELY WORK
WITH THEM TO HELP THEM GENERATE
AN APPROPRIATE PITCH IN THE
BIOMARKER DEVELOPMENT SPACE.
THE THIRD PROGRAM DOESN'T FALL
UNDER AN EXISTING MECHANISM BUT
BUILT AS A SPINOFF FROM CANCER
MOON SHOT A $220 MILLION PROGRAM
THAT WORKS WITH I THINK 16
PHARMACEUTICAL COMPANIES
FOCUSSED ON IMMUNO ONCOLOGY AND
DEVELOPMENT OF STANDARDIZED
ASSAYS AND BIOMARKERS FOR
DEVELOPMENT OF TRYING TO ADDRESS
COMBINATION THERAPY IN THE
DEVELOP OF THE ONCOLOGY SPACE
AND GOT EXTENDED ANOTHER FOUR
YEARS UNTIL 2026.
AND DURING THE PANDEMIC WE
FACILITATED AND HELPED BUILD THE
ACTIVE PROJECT IN THE
DEVELOPMENT OF THERAPEUTIC
INTERVENTIONS AND VACCINES AN
MASTER PROTOCOLS FOR BOTH
THERAPEUTICS AND VACCINES AND
THE INFRASTRUCTURE TO DO THAT.
WE DO SIMILAR WORK IN THE
POPULATION SPACE.
THIS WORK IS LARGELY FUNDED BY
PUBLIC FUNDING AND LARGE
FOUNDATION CONTRIBUTIONS.
AND YOU MAY BE FAMILIAR WITH OUR
LARGE SCIENTIFIC PROJECTS.
IN ADDITION TO THIS TYPE OF WORK
TO CELEBRATE AND TRAIN THE NEXT
GENERATION OF SCIENCE WE DO TRY
TO INTEGRATE THIS INTO OUR
INDIVIDUAL PROGRAM WORK.
ESPECIALLY MY BELIEF IS IF WE
DID A STUDY I WOULD LIKE TO DO
IT TO SHOW THE IMPACT OF
PARTICIPATING ON TEAM SCIENCE ON
INDIVIDUAL INVESTIGATOR'S CAREER
WE'D SEE A PROFOUND IMPACT IN
OUR MEDIATED DISEASE PROGRAM AS
WELL WITH SOME EARLY
INVESTIGATORS AND THE LEADERSHIP
SCHOLARS PROGRAM.
THERE'S ACTIVITY IN THE DISEASE
MAPPING AND DISEASE INTELLIGENCE
SPACE AND MAPPING THOSE TOGETHER
TO UNDERSTAND HOW TO ALIGN
PUBLIC AND PRIVATE INVESTMENT IS
AN ART, IT'S NOT A SCIENCE I'VE
LEARNED SINCE BEING AT THE
FOUNDATION.
IT'S INCREDIBLY IMPORTANT TO
REALIZE THE INVESTMENT WE'RE
MAKING WITH THE RESEARCH.
AND WE DESIGN TO BUILD THE
PROGRAMS COLLABORATIVELY.
WE ALL KNOW AND WE KNOW RESEARCH
PROJECTS ARE COMPLICATED BUT THE
HARD THINGS ARE WORTH DOING AND
IN THIS SPACE AS YOU KNOW IT
TAKES ALL PLAYERS TO DESIGN
SOMETHING THAT WILL BE USEFUL.
AFTER THAT WE DO BRING IN
PRIVATE INVESTORS AND WORK WITH
NIH COLLEAGUES TO IDENTIFY
PREFERABLY CROSS-INSTITUTE
INVESTMENT AND MANAGE THE
GOVERNANCE OF THE PROJECT AND
PROVIDE FINANCIAL STEWARDSHIP
VARYING FROM PROJECT TO PROJECT.
SOMETIMES WE TRANSFER A LARGE
AMOUNT TO THE NIH FOR ISSUING OF
AWARDS.
SOMETIMES WE ISSUE CONTRACTS
DIRECTLY.
IT DEPENDS ON THE NEEDS OF THE
PROJECT.
THE TOPIC TODAY IS ALSO WE
MONITOR THE PROGRESS OF THE
PROJECT AND HAVE A STRONG
IMPACT.
I'LL TALK A LITTLE BIT ABOUT
IMPACT AND HOW WE VIEW IMPACT A
LITTLE BIT DIFFERENTLY.
SO JUST AN EXAMPLE WITHIN MY
PORTFOLIO AND THERE ARE MANY
EXAMPLES I COULD USE BUT THIS IS
ONE I'M INTIMATELY FAMILIAR WITH
WHICH IS OUR AMP AIM.
THIS IS A SUCCESSOR PROJECT AND
THIS IS CHAIRED BY NAIMS AND
LUPUS RESEARCH ALLIANCE WE I
HAVE TO HAVE REPRESENTATION AT
THE HIGHEST LEVEL OF LEADERSHIP.
THIS IS A SUCCESSOR.
ONE OF OUR ORIGINAL AMPS
FOCUSSED ON RHEUMATOID ARTHRITIS
AND LUPUS, THAT REALLY FOCUSSED
ON DISEASE DECONSTRUCTION.
I IT WAS PIONEERED IN THE SINGLE
CELL TECHNOLOGY SPACE AND LOOKED
AT WHAT WAS HAPPENING WITHIN THE
CELLS AND TARGETS FOR THERAPIES
AND MOLECULAR CLASSIFICATION OF
DISEASE.
YOU SEE THIS IN REPRESENTATION
AT AMERICAN COLLEGE OF
RHEUMATOLOGY MEETING AND THE
PRESENTERS FROM THE
COLLABORATIVE AND AN AREA THAT
DOESN'T FREQUENTLY SEE THEM.
IT'S MORE THAN THAT.
IT'S THE FREQUENCY IN BIO
SCIENCE AND CHALLENGING DISEASES
TO LOOK AT STANDARD OPERATING
PROCEDURES THAT EXTEND FROM
ACADEMIA TO INDUSTRY AND THE
BRIDGE BETWEEN THE TWO SO WE CAN
DO CROSS-SITE STUDIES IN A
MEANINGFUL WAY.
IT IS REALLY UNDERSTANDING HOW
TO DO THE TECHNOLOGIES AND
FREQUENTLY EXPENSIVE
TECHNOLOGIES AND TAKE ADVANTAGE
OF THEM AND BIO STRATEGIES.
AND AMP AIM IS A $70 MILLION
INVESTMENT WE'RE HALFWAY THROUGH
AND EXPANDED THE DISEASE FOCUS
TO FOCUS ON PSORIATIC SPECTRUM
AND SJOGREN'S DISEASE FOCUSSED
ON MORE DISEASE RECONSTRUCTION
AND NEIGHBORHOODS AND PATHOLOGY
AND STATE OF THE ART TECHNOLOGY
TO UNDERSTAND THE MOLECULAR
UNDER PINNINGS OF THE DISEASES A
FOCUS ON EARLY DISEASE AS WELL
AS CROSS DISEASE STUDIES WHICH
IN THE AUTO IMMUNE SPACE IS
INCREDIBLY IMPORTANT.
WE'VE HAD TO GO BACK TO THE
DRAWING BOARD AFTER FAILED
CLINICAL TRIAL TO LOOK AT HOW WE
UNDERSTAND THESE DISEASES.
WE'RE ABLE TO DO PILOT STUDIES
AND I DO NOT HAVE MY Ph.D.
I WROTE MY UNDERGRADUATE THESIS
AND DID MASS SPEC ANALYSIS.
IT'S SO EXCITING TO SEE THE
TECHNOLOGIES THE ABILITY TO DO
SPECIAL SEQUENCING AND LAYER
OTHER TECHNOLOGIES ON TOP OF
THAT IS GAME CHANGING AND IT'S
EXCITING TO BE INVOLVED IN THIS.
AND AS YOU CAN SEE FROM THE
SLIDE THE HUGE RANGE OF SITES
INVOLVED.
THERE'S DISEASE FOCUSSED
MULTI-INSTITUTE TEAMS AND
SYSTEMS BIOLOGY AND FORCES AS
WELL.
AND THE FINAL PROJECT I WANT TO
TOUCH ON IS A PROJECT IN THE
DESIGN PHASE CALL SYSTEMS
BIOLOGY OF INFORMATION.
AND THIS TAKES ADVANTAGE OF THE
INVESTMENT TO CREATE USEFUL
ANALYSIS AND LONGITUDINAL
CLINICAL INFORMATION GENERATED
BY THOSE PROGRAMS AND THEY AIM
TO HAVE PUBLICALLY AVAILABLE
ACCESSIBLE DATA SETS IN AMP AIM
ACCESSIBLE IN THE PORTAL HOSTED
BY NIAMS.
AND THE INFRASTRUCTURE PIECE AND
THEN THE SCIENCE WE CAN DO WITH
IT.
AND THERE'S A PARALLEL PROJECT
FUNDED BY THE COMMON FUND.
THESE ARE INTERYALLY -- INTEGRAL
PROGRAMS DESIGN TO UNDERSTAND
HOW DIFFERENT DATA SETS CAN WORK
TOGETHER.
AND THIS JUST GIVES A SENSE OF
THE EARLY DATA SETS AVAILABLE
AND ALZHEIMER'S DISEASE,
PARKINSON'S DISEASE AND AMP AIM
AND SLE AS WELL.
THAT MODEL IS BUILD TO BE USED
WITH OUR FUTURE AMP DATA SETS AS
WELL AS OTHER OUTSIDE DATA SETS.
AND I BELIEVE THAT IS IT FOR ME.
THANK YOU.
>> THANK YOU VERY MUCH.
NEXT ON OUR LIST WE HAVE
REPRESENTATIVES FROM NIDDK, WE
HAVE DR. CHRISTINE MARIC-BILKAN,
DR. SUSAN MENDLEY, DR. DEEPAK
NIHALANI AND DR. IVONNE SCHULMAN
AND ALLOW THEM TO GIVE YOU THEIR
INDIVIDUAL TITLES IF I MAY.
>> I'M DEEPAK NIHALANI FROM THE
U.S. DIVISION OF THE NIDDK AND
WOULD LIKE TO THANK THE
ORGANIZERS FOR INVITING US FOR
THE PRESENTATION.
I'LL BE GIVING THE PRESENTATION
ON BEHALF OF MY COLLEAGUES WHO
ARE ALSO HERE WITH US AND
DR. SHULMAN THIS PRESENTATION
WE'LL DISCUSS THE PROGRAM THAT
CATERS TO DIVISION SUPPORT AND
PROVIDING SUPPORT TO THE
HEMATOLOGIC DISEASES.
AS THE NEEDS OF THE SCIENTIFIC
COMMUNITIES CHANGES IT WAS
DES
DECIDED THE PROGRAMS NEEDED TO
CHANGE AND DEVELOP AND SHARE
RESOURCES WITH THE COMMUNITY
THAT WILL HELP ADVANCE SCIENCE
AND ADDRESS GAPS IN
UNDERSTANDING AND AMELIORATION
OF KIDNEY AND OWE LOGIC DISEASES
AND PROMOTE NEW TALENT AND
DEVELOPMENT AND EXPANSION OF THE
KIDNEY WORKFORCE.
AND REALIZING THE INDIVIDUAL
RESEARCH CENTERS WERE SET UP
THROUGH DIFFERENT FUNDING
MECHANISMS AND THEY SUPPORT THE
FORMATION OF SPECIALIZED CENTERS
THAT FOCUSSED ON SERVING THE
PROGRAM GOALS AND DISEASES.
THE MAJORITY OF THE PROGRAMS ARE
CONGRESSIONALLY MANDATE AND
DISEASE SPECIFIC.
THESE ARE SOME OF THE KEY POINTS
THAT WERE REALIZED TO BE
INCLUDED IN THIS TRANSITION OF
THE CENTERS INTO THE CONSORTIUM.
WE IMPROVED TO THE COORDINATION
AND ADDRESS THE CHANGING
LANDSCAPE OF SCIENCE AND
HARMONIZE THE CENTERS.
IN ADDITION TO THESE THERE ARE
OTHER KEY FACTORS THAT WERE ALSO
PLAYING A PART IN REALIZING THE
NEED TO CHANGE THE PROGRAMS
WHICH WERE OUTREACH AND THE NEED
FOR INTERDISCIPLINARY SCIENCE
AND NEW RESOURCES AND TOOLS AND
IDENTIFICATION OF NOVEL
THERAPIES.
THEREFORE THEY WERE TRANSLATED
TO A NATIONWIDE EFFORT TO
ADDRESS RESEARCH.
THESE ARE CONGRESSIONALLY
MANDATED PROGRAMS AND ALSO SOME
OF THEM ARE DISEASE SPECIFIC.
THE FIRST ONE THE KIDNEY DISEASE
CENTERS IS ONE OF THE OLDEST
PROGRAMS OF THE GRANT CENTERS
AND INITIALLY ESTABLISHED IN
1986 BUT HAS NOW BEEN
RE-ESTABLISHED AS A PROGRAM
WHICH IS THE ALLIANCE
ESTABLISHED NOW 2003 AND THE
CONSORTIUM ESTABLISHED IN 2019
AND EACH OF THESE CENTERS FOCUS
ON ADVANCING RESEARCH IN THEIR
RELEVANT AREA OF RESEARCH WHICH
MAY INCLUDE MANY AREAS AND A
VARIETY OF RESOURCES INCLUDED
STATE OF THE ART TOOLS AND
DEATHS AND EXPERTISE ARE OFFERED
THROUGH THE PROGRAMS AND
CONSORTIA AND HELP THE
ADVANCEMENT OF DISEASES.
THE PURPOSE FOR THE PROGRAMS ARE
TO ENHANCE SHARING AND
DEVELOPMENT OF SPECIALIZED
RESOURCES THAT HELP SUPPORT THE
RESEARCH COMMUNITY IN ADVANCING
SCIENCE.
THEY ALSO ESTABLISH THE
FRAMEWORK TO INCREASE
COLLABORATION AND COORDINATION
AMONG RESEARCH SCIENTISTS,
CLINICIANS AND THE NIH THAT
HELPS PROMOTE THESE BASIC AND
CLINICAL RESEARCH AND NEW
TREATMENT AND PREVENTION.
THE PROGRAM ALSO HELPS DEVELOP A
COMMUNITY THAT ATTRACTS SCIENCE
AND SCIENTIFIC AND DIVERSE AND
DEVELOPMENT AND IMPLEMENTATION
OF DEVELOPED PROGRAMS THAT HELP
PROMOTE YOUNG SCIENTIST AND
EMERGING INVESTIGATORS.
SO NEXT I'M GOING TO BRIEFLY
DISCUSS ABOUT THE MEETING WHICH
IS OUR INTERAGENCY COORDINATING
COMMITTEE.
THIS IS ALSO MANDATED BY
CONGRESS IN 1987 AND IS GOING
THE PRIMARY GOAL FOR THE
COMMITTEE IS TO INTEGRATE
COOPERATION AND COLLABORATION
AMONG ALL THE AGENCIES THAT ARE
INVOLVED IN THE RESEARCH AND
ACTIVITIES AND RECOGNIZES THE
NEED FOR RESPONSE AND SOME OF
THE OBJECTIVES FOR INCLUDE
FEEDBACK ON PLANS AND IDEAS TO
HELP MOVE THE NEEDLE ON KIDNEY
DISEASES.
THIS IS A GRAPHIC REPRESENTATION
OF A BIRD'S-EYE VIEW OF CDC AND
NIDDK AND HIGHLIGHT THE
IMPORTANCE OF INTERAGENCY
COMMUNICATION.
TO THIS MEETING IT BRINGS THE
FACETS TOGETHER.
I HAVE AN ORGANIZER OF THE
MEETING IS AVAILABLE TO ANSWER
QUESTIONS.
THANK YOU.
>> WE'LL HAVE QUESTIONS AT THE
END AND ONE MORE SET OF
PRESENTATIONS FRE THIS COPD
ACTION PLAN, MR. JAMES KILEY AND
DR. NEYAL AMMARY-RISCH.
>> SORRY WE'RE NOT ABLE TO BE IN
PERSON BUT HERE TO HELP PRESENT
SOME OF THE THINGS WE'RE DOING
IN THE LUNG AREA OF THE
INSTITUTE THAT MAY BE RELEVANT
TO THE COMMISSION'S WORK.
IT'S ABOUT TO COME BACK AND TALK
ABOUT THE LUNG AREA.
THIS IS IN TERMS OF OUTPUT AND
PROGRESS.
I'M NOT GOING TO DWELL ON THIS
BUT I THOUGHT WE SHOULD ANCHOR
YOU ALL IN TERMS OF WHAT THIS IS
ALL ABOUT.
SO THIS IS ABOUT CHRONIC
OBSTRUCTIVE PULMONARY DISEASE.
PROBABLY MOST IN THE ROOM IF NOT
ALL RECOGNIZE THIS IS THE FOURTH
LEADING CAUSE OF DEATH IN THE
COUNTRY AND IT AFFECTS ABOUT 17
MILLION PEOPLE, 1 IN 5 AMERICANS
OVER 35 AND THE LEADING CAUSE OF
DEATH AND DISABILITY AND HIGH
MORTALITY RATE OF 150,000
AMERICANS EVERY YEAR AND SEE
THOSE DIAGNOSED WITH COPD AND SO
FORTH.
IT'S NOT COPD EXACTLY THAT I
THINK YOU WANT TO HEAR ABOUT SO
MUCH.
SO WE'RE GOING TO NOW PIVOT AWAY
FROM THE DISEASE ITSELF BUT TELL
YOU A LITTLE BIT ABOUT WHY WE
NEED AN ACTION PLAN.
IT MAY BECOME KIND OF OBVIOUS TO
YOU WHEN HAVE YOU A DISEASE OF
THIS MAGNITUDE AND SCOPE.
SO WHAT IS THE COPD NATIONAL
ACTION PLAN?
THIS IS THE FIRST EVER BLUEPRINT
THAT WAS DEVELOPED BY A WIDE
RANGE OF STAKEHOLDERS IN A
MULTI-FACETED WAY IT UNIFY THE
FIGHT AGAINST THIS DISEASE.
IN MANY WAYS THOUGH IT HAS A
VERY HIGH MORTALITY RATE AND
PREVALENCE, IT ALSO IS A BIT
FRAGMENTED IN TERMS OF HOW THE
COMMUNITY IS APPROACHING IT
WHETHER FROM A DISEASE OR
ADVOCACY OR IN TERMS OF GENERAL
EDUCATIONAL MATERIAL.
THIS PLAN WAS REALLY AND
COMPREHENSIVE FRAMEWORK FOR
ACTION ON THOSE AFFECTED BY THE
DISEASE AND THOSE WHO CARE FOR
THOSE AFFECTED BY THE DISEASE
AND AT THE SAME TIME TO TRY TO
AGAIN REDUCE THE BURDEN, BRING
ABOUT A BIT MORE COORDINATION
AND TRY TO HIT ALL THE DIFFERENT
SEGMENTS ONE WOULD NEED TO
ADDRESS IN TERMS OF REALLY
TAKING ON COPD IN A
COMPREHENSIVE WAY.
THIS IS WHAT THE ACTION PLAN IS
ALL ABOUT.
DEVELOPED THROUGH A
COLLABORATIVE PROCESS ACROSS THE
COPD COMMUNITY WORKING TOGETHER
TO PROVIDE PATIENT-CENTEREDNESS
AND LOOKS AT HEALTH CARE
DELIVERY AND PRACTICE AND
INFORMS POLICY AND ADVOCACY AND
BUILT AROUND FIVE GOALS AND 36
OBJECTIVES.
NOW, I'M NOT GOING TO GO THROUGH
EVERY SINGLE ONE BUT SKIM OVER
THE TOP AND LET YOU SEE A
GLIMPSE OF WHAT IS IN THIS
ACTION PLAN.
IT IS ONLINE.
IT'S ALSO AVAILABLE PROBABLY IN
HARD COPY IF YOU WANT TO SEE ALL
OF THE ELEMENTS OF IT.
IT'S VERY WELL DONE AND
COMPREHENSIVE AND IT'S AN EASY
READ FRANKLY.
THOUGH IT MAY BE A LITTLE BIT
INTIMIDATING WHEN YOU SEE THE
SIZE IT'S PRETTY QUICK TO GO
THROUGH AND GET A SENSE WHERE
THE GAPS AND NEEDS ARE.
SO THIS ALL STARTED THEN IN
FEBRUARY OF 2016 WHILE WE GOT
THE DIRECTIVE FROM CONGRESS A
BIT BEFORE THAT BUT BEGAN TO PUT
OUR HEADS TOGETHER IN CREATING
THE NATIONAL ACTION PLAN.
SO ONE OF THE THINGS WE SAID WE
WOULD DO IS TO SET THE
FOUNDATION FOR THE PLAN AND THE
WAY WE WENT ABOUT THAT WAS WE
CREATED WHAT WE CALL THE TOWN
HALL MEETING.
THIS BROUGHT TOGETHER IN FACT IN
A ROOM BECAUSE THIS IS
PREPANDEMIC MORE THAN ABOUT 200
INDIVIDUALS ATTENDEES, PATIENTS,
CAREGIVERS, THE WHOLE RANGE,
INDUSTRY LEADERS, ACADEMIC
LEADERS, 14 FEDERAL PARTNERS ALL
PARTICIPATED.
THIS WAS A PRIVATE SECTOR AND
GOVERNMENT LED EFFORT.
IT WAS A HEAVY LIFT.
IT WAS A HUGE UNDERTAKING.
A BIG SHOUT OUT TO NEYAL AND
OTHER INSTITUTE OFFICE OF POLICY
AND STRATEGIC OPPORTUNITIES TO
REALLY SAY WE REALLY HAD TO
BRING TOGETHER ALL THE RESOURCES
WITHIN NHLBI TO PULL THIS OFF.
AND WHAT HAPPENED DURING THIS
TOWN HALL MEETING YOU SEE A
GLIMMER OF THE ATTENDEES, WE
BROKE INTO SIX BREAKOUT GROUPS
AND THEY WERE BASED AROUND THE
ORIGINAL ACTION PLAN GOALS.
AND THIS IS HOW WE CAME UP WITH
18 RECOMMENDATIONS FROM THESE
SIX DIFFERENT GROUPS.
I'M NOT GOING TO GO INTO ALL THE
DETAILS OF THIS BUT WANTED TO
LET YOU SEE THE TIME LINE THAT
WENT INTO THE DEVELOPMENT OF THE
PLAN.
AS I MENTIONED PRE 2016 WE GOT
THE CONGRESSIONAL DIRECTIVE.
IN '16 WE DEVELOPED THE FIRST
DRAFT OF THE PLAN WITH ALL THE
INPUT AND SOLICITED FEEDBACK
FROM THE COPD TOWN HALL
ATTENDEES.
THE PLAN WAS THEN REVISED LATE
IN 2016 AND REVIEWED AGAIN IN
LATE IN 2016 AND WENT THROUGH
FINAL REVIEW AND GOVERNMENT
CLEARANCE REQUIRED TO PUT OUT A
GOVERNMENT DOCUMENT IN APRIL
RELEASED IN MAY.
THEY TAKE TIME.
I DOESN'T HAPPEN OVERNIGHT.
IT'S A CLEAR TAKEAWAY.
YOU HAVE TO BE PATIENT AND LET
THE PROCESS PLAY OUT.
BUT YOU GET A SUPERB PRODUCT AT
THE END.
HERE ARE THE FIVE MAJOR GOALS TO
EMPOWER PEOPLE WITH COPD AND
FAMILY AND CAREGIVERS TO
RECOGNIZE AND REDUCE THE BURDEN.
THE SECOND WAS DO APPROVE
PREVENTION, TREATMENT AND
MANAGEMENT OF COPD AND ENHANCE
THE QUALITY OF CARE ACROSS THE
HEALTH SPECTRUM.
THIRD IS TO COLLECT, ANALYZE AND
REPORT AND DISSEMINATE PUBLIC
HEALTH RELATED DATA THAT WOULD
DRIVE CHANGE AND TRACK PROGRESS.
FOUR WOULD BE INCREASE AND
SUSTAIN THE RESEARCH TO BETTER
UNDERSTAND THE DISEASE,
PATHOGENESIS UP TO MANAGEMENT
AND THEN TRANSLATE NATIONAL
POLICY AND PROGRAM
RECOMMENDATIONS INTO RESEARCH
AND PUBLIC HEALTH ACTIONS.
WHAT I HOPE YOU CAN SEE IS IT
TOUCH EVERY SECTOR OF SOCIETY.
IT'S NOT JUST OF AN AT
RESEARCHERS.
NHLBI HAS A ROLE BUT IT'S
PRIMARILY IN GOAL FOUR IN SOME
OF FIVE MAYBE WHEN YOU LOOK AT
THREE YOU SEE A LOT OF CDC IN
THERE YOU CAN SEE WHERE A
DIFFERENT GOVERNMENT AGENCY AND
STAKEHOLDER GROUP WHETHER A
FOUNDATION OR FAMILY RUN
ORGANIZATION OR ANY OTHER SECTOR
THAT TOUCHES INDIVIDUAL PATIENTS
WITH THE DISEASE CAN FIND
THEMSELVES IN THE PLAN.
AND WE STARTED IMPLEMENTATION OF
THE PLAN AND THAT DEPENDS ON THE
ENTIRE COPD COMMUNITY.
OVER AND OVER AGAIN IN ALL KINDS
OF PUBLIC MEETINGS HAVE
INDICATED THAT THIS IS NOT JUST
A PLAN FOR NHLBI.
THIS IS NOT A PLAN FOR NIH.
THIS IS NOT JUST ONE AGENCY OR
ORGANIZATION.
IT'S THE ENTIRE STAKEHOLDER
COMMUNITY THAT NEED TO DRIVE THE
CHANGE AND SUPPORT THE PROGRAMS
AND ACTIVITIES TO HELP REDUCE
THE BURDEN OF THE DISEASE.
WE USE THE METAPHOR RELATED TO
MOVE THE NEEDLE.
WE SAID THAT EARLY ON.
I'VE USED THE ANALOGY IF YOU
LOOK AT THE GAS TANK IN YOUR CAR
WHERE ARE WE WHEN WE LOOK AT THE
NEEDLE.
MAYBE WE'RE A QUARTER FULL BUT
WANT TO GET THE TANK TOPPED OFF
AND SEE OUR NED -- NEEDLE GET
TO FULL AND A PUZZLE LOOKING
FORMAT WHERE IT INVOLVED
DIFFERENT ORGANIZATIONS AND THEY
ALL COME TOGETHER AND ALL
CONTRIBUTE IN SOME WAY TO MAKING
TO ACHIEVE THOSE GOALS.
AND WHAT WE TRY TOE DO IS
OUTLINE THE FIVE GOALS AND PUT
THEIR SCHEMATIC OF WHERE THE
DIFFERENT ORGANIZATIONS PLUG
INTO DIFFERENT GOALS SO YOU CAN
SEE WHERE YOU ARE ON THE BIGGER
EFFORT.
HOPEFULLY PEOPLE CAN USE THIS IN
THEIR OWN ORGANIZATION WHETHER
RESEARCHER OR HEALTH
PROFESSIONAL OR POLICY MAKER OR
ANOTHER CONCERNED INDIVIDUAL.
AND THERE IS A PLACE WHERE EACH
INDIVIDUAL PERSON, ORGANIZATION
OR ENTITY CAN PLUG IN AND TAKE
AN ACTIVE ROLE IN IMPLEMENTING
THE PLAN.
I'LL TURN IT OVER TO NEYAL TO
FINISH UP HERE.
NOT ONLY DID WE HAVE TO DEVELOP
THE PLAN AND COME UP WITH A WAY
TO REPORT AND DEVELOP A TRACKING
PROCESS.
THIS MAY BE A WAY TO LOOK AT
THIS IN TERMS OF HOLDING
EVERYONE ACCOUNTABLE.
IF EVERYONE FEELS THEY HAVE A
ROLE IN THE DEVELOPMENT OF THE
PLAN AND RECOMMENDATION THEY
ALSO SHOULD HAVE A ROLE IN
IMPLEMENTING IT BUT THEN WE NEED
A REPORT CARD.
SOME WAY TO SAY WHAT DID YOU DO
TO HELP MOVE THE NEEDLE.
THIS IS THE PART OF THE
PRESENTATION WE'RE IN NOW.
WE'RE NOW RECOMMENDATION
RECOMMENDING THE PLAN.
WE WANTED TO FIND A WAY TO TRACK
THIS BUT IT'S ALSO PART OF THE
GOAL AND ONE OBJECTIVE IS TO
TRACK THROUGH REPORTING.
AROUND WE WERE THINKING ABOUT
HOW DO WE DO THIS OR MONITOR
THIS?
WE HAD A LOT OF DISCUSSIONS
INTERNALLY AND WITH OUR COPD
STAKEHOLDER COMMUNITY TO TALK
ABOUT THIS AND EXPLORED SOFTWARE
THAT WAS CUSTOMIZEDAB ABAB ABAB
CUSTOMIZABLE THAT'S OPEN TO THE
COPD COMMUNITY THEY CAN COME TO
AND ADD THEIR PROJECTS AND
RESOURCES AND EVEN WITH THIS
CONTINUE TO BE ESSENTIAL.
WE PUT TOGETHER A FIRST DRAFT OF
WHAT ARE THE THINGS WE WANT TO
TRACK AND WORK WITH THE
COMMUNITY TO TRACK ALL THE
FIELDS AND WHAT ARE THINGS WE
WANT TO KNOW ABOUT THE GOALS AND
OBJECTIVES AND SOMETHING EASY
AND SIMPLE AND TAKES FIVE
MINUTES FOR AN ORGANIZATION TO
INPUT INFORMATION.
WE DEVELOPED THIS COMMUNITY
ACTION TOOL TO HELP WITH
ACTIVITIES AND WHICH GOALS AND
OBJECTIVES ARE GETTING ACTIVITY
AND AN OPPORTUNITY FOR THE COPD
COMMUNITY TO COLLABORATE AND IF
THERE'S THOUGHT OF A PROGRAM FOR
RURAL PROVIDERS THEY CAN CONNECT
WITH FOLKS TO LEARN FROM EACH
OTHER AND ISSUE PERIODIC REPORT
CARDS AND WHERE WE'RE MAKING
PROGRESS AND WHERE WE NEED TO
SEE THE NEEDLE MOVE.
AND ONE OF THE THINGS WE KNOW
ABOUT IS IT CAN ONLY BE AS
SUCCESSFUL AS WE MAKE IT AND THE
DATA'S ONLY AS GOOD AS THE DATA
IN IT.
THERE'S MOMENTUM AND EXCITEMENT
WHEN WE BROUGHT THE COMMUNITY
TOGETHER.
WE SAID IF WE BUILD IT WILL YOU
COME AND THEY SAID YES AND TALK
ABOUT POLICIES THAT NEED TO BE
MADE.
THERE'S MOMENTUM BEHIND IT.
WE HAVE ABOUT 105 ENTRIES
RIGHT NOW AND THE TYPES OF
ACTIVITIES LOOKING AT THE
ACTIVITY WHETHER HEALTH
PROFESSIONAL OR ACADEMIC
ORGANIZATION FOR FEDERAL OR
STATE AGENCIES.
IS IT FOR CAREGIVERS?
POLICY MAKERS?
JUST THE PUBLIC OR HEALTH CARE
PROVIDERS, AT RISK POPULATIONS?
THE ACTIVITIES ARE TAKING PLACE
AND IS IT A WORKPLACE THING FOR
CLINICAL SETTING TYPES OF
ACTIVITIES?
IS IT ADVOCACY OR TRAINING OR
CME FOR HEALTH CARE PROVIDERS
AND BREAK IT DOWN FURTHER INTO
THE SPECIFIC POPULATIONS BEING
REACHED.
ALSO GEOGRAPHIC IN TERMS OF
NATIONAL IN SCOPE OR WHAT YOU'RE
DOING REGIONAL OR LOCALIZED AND
LIKE THE REACH UP TERMS OF HOW
INFORMATION IS GETTING OUT AND
TO WHERE.
WE'LL BE HOLDING A MEETING IN A
FEW WEEKS IN NOVEMBER TO BRING A
COPD STAKEHOLDER COMMUNITY BACK
TOGETHER TO REENERGIZE THEM AND
REMIND THEM THIS BELONGS TO
EVERYBODY IN THE COMMUNITY AND
THIS IS LIKE THE ONE SYSTEM THAT
WE HAVE FOR BEING ABLE TO TRACK
AND REPORT AND COME TO THE
WEBSITE AND THE GRAPHS ARE
DOWNLOADABLE TO PEOPLE CAN PULL
THEM INTO PRESENTATIONS AS THEY
ADVOCATE FOR IMPLEMENTATION OF
THE PLAN.
WE HAVE HARD COPIES OF THE PLAN
AND YOU'RE WELCOME TO LOG ON AND
YOU CAN SEE THE TYPES OF DATA
BEING COLLECTED IN THE COMMUNITY
ACTION TOOL.
THAT'S IT.
IF YOU HAVE QUESTIONS, HAPPY TO
ANSWER.
>> WE'LL NOW ENTER THE QUESTION
AND ANSWER PHASE AND TURN IT TO
SELEN.
>> THANKS TO OUR PANELISTS.
I WANT TO ACKNOWLEDGE IT'S VERY
DIFFICULT TO BOIL DOWN
EVERYTHING YOU'RE HOPING TO DO
INTO A SET OF SLIDES BUT IN
WATCHING COMMISSIONERS I CAN
TELL YOU SPARKED QUESTIONS.
MENTIONED EARLIER TODAY WE'RE
WORKING UNDER SIX COMMITTEES.
EACH COMMITTEE BROUGHT QUESTIONS
FOR YOU.
SOME HAVE CHANGED IN THE PROCESS
OF PRESENTATIONS.
A FEW OF OUR COMMITTEES WERE NOT
ABLE TO ASK QUESTIONS THIS
MORNING AND SO I'LL START WITH
THEM THIS AFTERNOON.
DO YOU HAVE A QUESTION?
>> I'M A CO-LEAD OF THE
COMMUNITY GROWTH COMMITTEE AND
MANY PARTNERS WITH MULTIPLE
COMPETING PRIVATE ENTITIES, CAN
YOU SPEAK TO HOW YOU MADE YOUR
WAY THROUGH THAT AND HOW YOU
PROTECT PROPRIETARY INFO AND
INCENTIVIZING THAT
COLLABORATION?
>> WE TRY TO FOCUS ON THE
PARTNERSHIPS.
THAT LINE CAN BECOME BLURRY
THESE DAYS AROUND THE USE OF
A.I. TECHNOLOGY AND OTHER
APPROACHES.
I WILL SAY WE DON'T PUT IN.
WE HAVE WE ARE CLEAR AT THE
BEGINNING OF OUR PROGRAMS THAT
COMPANIES ARE NOT TOO SHARE
PROPRIETARY INFORMATION WITHIN
OUR COMMITTEES.
WE DON'T WANT IT TO BE SHARED.
WE DO NOT PUT IN THE TYPES OF
NON-DISCLOSURE AGREEMENTS THAT
WOULD PUT PENALTIES UPON EITHER
THE FOUNDATION OR ANY OF OUR
PARTNERS.
THAT'S FOR UTILIZING THE
TECHNOLOGY.
THERE ARE OBVIOUSLY SOME
EXCEPTIONS THAT I WILL GIVE YOU
SOME EXAMPLES OF THE
COMPLEXITIES OF THIS.
SO THE BESPOKE GENE THERAPY
CONSORTIUM I TALKED ABOUT
EARLIER, OBVIOUSLY THOSE ARE
RARE DISEASE PROGRAMS WHERE THE
ABILITY TO COMMERCIALIZE GENE
THERAPIES FOR THOSE DRUGS IS NOT
VIABLE WITHOUT THIS TYPE OF
REGULATORY PATHWAY.
SO CERTAINLY AGAIN THERE'S AN
UNDERSTANDING THAT COMPANIES ARE
PARTICIPATING IN GENERATING THIS
KIND OF SHARED PATHWAY AND THAT
THERE COULD BE OBVIOUSLY
INTELLECTUAL PROPERTY THAT THEY
USE THAT INFORMATION TO GENERATE
INTERNALLY.
EQUIVALENTLY THOUGH WE HAVE A
DESIGN PROJECT THAT'S FOR
CELL-BASED THERAPIES.
THAT ONE IS PLORE -- MORE
COMPLICATE AND THIS IS A
QUESTION WE RUN INTO.
FOR SOME OF THESE BROADER USE
APPLICATIONS THAT DO HAVE
VIABILITY, HOW DO WE HANDLE
THAT?
WE HAVEN'T COMPLETELY FIGURED
THAT OUT YET.
WE'RE MOVING INTO A LITTLE BIT
OF A NEW WORLD HERE.
AND IN SOME CASES WHEN YOU'RE
TALKING ABOUT TRANSLATIONAL
SPACE, THE GOAL IS FOR PEOPLE TO
TAKE THESE THINGS AND DEVELOP
THEM FURTHER.
WE WANT PEOPLE TO BE
INCENTIVIZED TO DEVELOP DRUGS
AND TREATMENTS.
WE ARE IN THE PROCESS OF
NAVIGATING FOR THAT PROJECT.
BUT GENERALLY WE TRY TO STAY IN
THAT SPACE.
>> WOULD ANYONE ELSE LIKE TO
ANSWER THAT QUESTION OR SHOULD
WE MOVE ON?
ALL RIGHT.
CAN YOU HEAR FROM THE SCIENTIFIC
ADVANCEMENT COMMITTEE.
YOU ALL HAVE A NUMBER OF
QUESTIONS.
>> FOR THE NIDDK AND COPD ARM
BECAUSE WE DISCUSSED ONE OF THE
ISSUES WE ARE FACING AS A
COMMUNITY IS A, HOW TO INCREASE
THE NUMBER OF INVESTIGATORS
WORKING IN THE FIELD AND IN THE
CENTER.
THOUGH THERE WERE UPS AND DOWNS
FROM THAT EXPERIENCE.
AND THERE'S ONE APPROACH TO
FOLLOW AND TO HAVE THE
OPPORTUNITY TO HAVE CENTERS THAT
HAVE DIFFERENT ASPECTS OF THE
RESEARCH COULD BE A POSSIBILITY
TO ADDRESS THOSE ISSUES AND
AGAIN PROVIDE GRANTS, ETCETERA.
YOU INDICATED THIS WAS SET IN
DATA BUT HOW IS THE INITIAL STEP
CREATED FOR THE CENTERS HOW DID
IT HAPPEN?
THAT CAN BE USEFUL FOR US AS ONE
OF THE MECHANISMS HOW TO MOVE
FORWARD WITH THAT.
>> I'M TRYING TO PROVIDE ANSWERS
BUT YOU CAN PROVIDE MORE AND
INITIALLY AS I DISCUSSED IN MY
SLIDE THESE ARE CONGRESSIONALLY
MANDATED THROUGH THE FUNDING
MECHANI
MECHANISMS THERE WERE DIFFERENT
CENTERS AND THESE ARE INDIVIDUAL
CENTERS CONDUCTING INDIVIDUAL
RESEARCH ON THOSE PARTICULAR
DISEASE AREAS AND THERE ARE WITH
VIROLOGY CENTERS BUT THESE HAD
INDEPENDENCE AND THROUGH THE
SMALL GRANTS AND AT THE TIME WE
REALIZED THERE WERE MORE NEEDS
AND THE GOALS OF THE PROGRAMS
WERE NOT BEING MET AND THERE WAS
A CONCERTED EFFORT AND NIH
BECAME A PARTICIPANT IN THE
CENTERS TO ENSURE THE GOALS OF
THE PROGRAMS ARE BEING MET.
>> BE CAREFUL WHAT YOU WISH FOR.
>> THERE IS A MANDATE THAT THERE
BE KIDNEY RESEARCH CENTERS NAMED
AFTER GEORGE O'BRIEN AND POLY
CYSTIC KIDNEY DISEASE CENTERS.
THEY'RE MANDATED.
THEY'RE NOT FUNDED.
SO NIDDK FUNDS THEM.
THEY WERE PRODUCT YOU HAVE BUT
WE WERE CONCERNED MAYBE THEY
WERE TOO ISOLATED.
DEEPAK MENTIONED THE TRANSITION
FROM DIFFERENT CENTERS TO
CONSORTIA IN THE HOPE THEY WOULD
BE MORE EFFECTIVE BOTH IN
SHARING OF RESOURCES AND ALSO
UNDERTAKING PROJECTS THEY
OTHERWISE MAY NOT HAVE DONE ON
THEIR OWN.
THERE'S AWARD CENTER.
HAPPY TO ANSWER ANY OTHER
QUESTIONS.
>> IN THE END THE IT WAS FUNDED
FROM NIDDK?
>> YES, MONEY DID NOT COME FROM
CONGRESS.
>> CAN YOU BRING EXAMPLES HOW TO
BRING THEM TOGETHER.
DID YOU ASK THEM TO COLLABORATE
OR INITIATE S NONE WERE DOING ON
THEIR OWN AND HOW SUCCESSFUL WAS
THAT?
>> FIRST, WE EXPECTED INSTEAD OF
DESCRIBING DISCREET PLANS AS
THEY ARE HAD DONE IN THE PAST
ABILITY TO SHARE RESOURCES THEY
CREATED, THEY HAD TO HAVE A
MECHANISM FOR OUTSIDE SHARING.
MANY FELT THEY WERE SHARING BY
SHARING INTERNALLY AND BECAME
INSTITUTIONAL RESOURCES AND WE
WANTED THEM TO BE NATIONAL EVEN
INTERNATIONAL RESOURCES.
THE FUNDING REQUEST FOR
APPLICATIONS CHANGED.
THE REVIEW PROCESS CHANGED.
IT REFLECTED OUR EXPECTATION
THAT THE GROUPS WOULD ACTUALLY
MEET AND SHARE AND DEVELOP
MECHANISMS NOT JUST TO SHARE
WITHIN THEIR SMALL GROUP BUT TO
SHARE OUTSIDE.
THAT WAS A CULTURE CHANGE.
THE POLY CYSTIC DISEASE KIDNEY
CENTERS DID IT AND THE UROLOGY
CENTERS DID IT FIRST AND THE
POLY CYSTIC NEXT AND THE O'BRIEN
CENTERS WERE NEXT.
EACH HAS THEIR OWN PERSONALITY
BUT THEY MEET AND PART OF THEIR
TASK IS TO DESCRIBE WHAT THEY
CAN COVER THE RESOURCES THEY
GENERATED CAN BE SHARED EX TERM
-- EXTERNALLY.
WE ASKED THEM TO DEVISE
RESOURCES THAT WOULD BE OF
GREATER VALUE TO THE COMMUNITY
AND JUSTIFY THEY'RE GENERALLY
USEFUL NOT JUST TO THEIR
LABORATORY OR INSTITUTION.
>> THERE'S A CONGRESSIONAL
MANDATE BUT DO THEY EXIST IN THE
ABSENCE OF THE MANDATE.
>> THAT'S GOING BEFORE MY TIME.
I COULD NOT ANSWER THAT
QUESTION.
>> THERE WAS A REQUEST FROM
PEDIATRIC KIDNEY DISEASE HAVE A
SIMILAR VENUE BUT DO NOT HAVE
THE SAME CONGRESSIONAL MANDATE.
>> I'LL ASK A DIFFERENT WAY
THEN.
>> IF THAT COMES FROM THE
CENTERS NOW -- SO WE'RE THINKING
ABOUT SUSTAINABILITY OF ANYTHING
WE CREATE.
SO THERE WAS AN ORIGINAL
CONGRESSIONAL MANDATE WITHOUT
FUNDING THAT GOT THINGS STARTED.
IS IT STILL IN THE ABSENCE OF
CONGRESSIONAL MANDATE IS IT
SUCCESSFUL ENOUGH?
>> THERE'S DIABETES AND DIGEST
IR DISEASE CENTER PROGRAMS.
THEY'RE SEEN AS USEFUL VENUES
FOR CREATING SCIENTIFIC
ADVANCES.
AND IF WE DIDN'T HAVE TO DO
THAT, WOULD WE DO THEM?
I DON'T THINK WE'D WALK AWAY.
THEY HAVE DEMONSTRATED VALUE.
>> THEY HAVE BEEN SERVING THE
COMMUNITY WELL.
>> WE APPRECIATE THE DIALOGUE.
>> I'VE BEEN PART OF THE
DIFFERENT STAGES AND THERE WERE
SOME FLAWS.
AND I THINK IT'S A GREAT
RESOURCE.
THAT'S PROGRESS.
>> AND WE OPERATE --
>> TOOK MONEY PART OF A P30 OR
P50 YOU ADDED MONEY OR THE
BUDGET STAYED THE STATEMENT OR
THE APPROACH CHANGED?
>> LET'S START BY SAYING WE
CHANGED THE APPROACH FIRST
BECAUSE WE FELT THE INDEPENDENT
CENTERS WERE NOT AS BENEFICIAL
AS IF THEY WERE INVITED INTO OR
PLACED INTO A CONSORTIUM TYPE
STRUCTURE.
THE FUNDS THAT BECOME AVAILABLE
HAVE MORE TO DO WITH THE NIDDK
OVER ALL BUDGET AND THE BUDGET
FOR KIDNEY DISEASE AND
HEMATOLOGIC DISEASE.
IT'S NOT A CASE OF ANY ONE
CENTER HAD A FOOTPRINT OF MONEY.
THERE'S A COMMITMENT MADE BY
NIDDK FOR EACH OF THESE CENTERS
AND PROGRAMS.
IN UROLOGY AND DIGESTIVE
DISEASES LOU THE DECISION IS
MADE COMES OF THE OVER ALL
COMMITMENT TO THE NIDDK MAKES TO
RESEARCH IN ALTHOUGH DISEASE
AREAS RECALLING THE RO1 RESEARCH
PROGRAM PROJECT PAY LINE IS A
BIG AMOUNT OF MONEY.
THAT'S WHERE MOST THE FUNDING
GOES.
>> I HAVE A QUESTION OR ANY OR
ALL THE PANELISTS.
ONE QUESTION WE'VE BEEN
STRUGGING WITH IS SHOULD
LYMPHATICS HAVE A CENTRAL HOME
AND IF THE ANSWER IS YES, A
SECONDARY QUESTION IS SHOULD
THAT BE WITHIN THE NIH OR
OUTSIDE THE NIH?
SO, I'D LIKE TO ASK ON A
HYPOTHETICAL BASIS THE PROS AND
CONS OF THE NIH OR IN
PARTNERSHIP WITH THE NIH BUT
FROM THE OUTSIDE OPPOSED TO AND
THE INSTITUTE.
>> IT'S A DIFFERENT ISSUE.
THERE'S QUESTIONS I COULD SEE
GOING INTO THIS IN TERMS OF THE
TYPE OF SCIENCE YOU'RE LOOKING
TO DO AND THE PROXIMITY OF THE
RESEARCH QUESTIONS AND DESIGNS
TO THE KINDS OF THINGS NIH DOES.
IN SEARCH WAY AS WELL AS THEN
THINKING ABOUT THE AGENDA AND
HOW THE AGENDA WILL GO.
AND THERE'S CENTRIC SUPPORTS AS
WELL AS THE CROSS-CUTTING NATURE
OF THE INSTITUTES AND CENTERS.
OBVIOUSLY THERE'S 27 INSTITUTES
AND CENTERS BRINGING THEM
TOGETHER HAS CHALLENGES TOO.
SO I THINK IT'S JUST SOMETHING
TO THINK ABOUT PARTICULAR GOALS
THAT YOU'RE GOING FOR AND THE
SUSTAINABILITY THAT YOU'RE
LOOKING FOR.
WHERE THINGS MIGHT GO AND WITH
PCORI WHERE THERE WAS A QUESTION
SET UP TO BE INDEPENDENT THAT
WAS REALLY TRULY ABOUT THE
DESIRE AND CONVERSATIONS WING
OUT THE WERE IMPORTANT TO BE
ABOUT TRANSLATION OF IMPACT AND
MOVE BEYOND THE BASIC RESEARCH
AND THINK ABOUT THOSE THINGS SO
IS THE AGENDA IS SHAPED IN SOME
FUNDAMENTALLY DIFFERENT WAYS.
THEY'RE NOT APPLICABLE TO NIH
AND WE'RE HOPING TO SHARE
KNOWLEDGE HOW TO DO THIS AND
THERE'S DIFFERENT
INFRASTRUCTURE.
IT'S AN IMPORTANT QUESTION TO
THINK WHAT THE SCALE AND FUNDING
WOULD BE AND SUSTAINABILITY
QUESTIONS ARE.
>> CAN I ASK, COULD YOU IDENTIFY
A PRICE THAT WAS PAID FOR -- I
HEAR THE BENEFITS IS THERE
ANYTHING ON THE NEGATIVE SIDE WE
ON THE NIH COULD HAVE DONE?
>> I WASN'T THERE DURING
START-UP SO I CAN'T SPEAK IT THE
SPECIFIC ISSUES IN THAT REGARD I
WAS AT NIH BEFORE I WENT TO
PCORI SO I CAN RELATE A LITTLE
BIT IN HOW THINGS ARE DIFFERENT.
UP WITH REALITY IS A STAND ALONE
FUNDING QUESTION FROM CONGRESS
AND SEE OTHER THINGS WHICH IS A
DIFFICULT QUESTION OR COULD GO
AWAY.
THAT'S SOMETHING WE MUST ALWAYS
BE MINDFUL OF WITH THE
STRUCTURES THERE.
OTHER ISSUES WILL BE
CONNECTIVITY TO SOME OF THE
OTHER SOURCES.
THERE'S A COST OF START UP TIME
TO ANY ORGANIZATION AND THERE'S
A COST IN TERMS OF ACTIVATION.
THERE'S DIFFERENT MODELS OUTSIDE
OR INSIDE AND OTHER RECENT
EXAMPLES TO BE LOOKED AT IN THAT
REGARD AND I THINK DIFFERENT
WAYS TO DO THEM.
THE LAST TOPIC WAS INTEREST IN
MORE INVESTIGATORS FOCUSSED ON A
DISEASE AREA AND CENTER FOR
EXCELLENCE AND SUSTAINABILITY
AND CONSISTENCY OF FUNDING IS A
HUGE PART OF THAT AND THE
SIGNALLING OF CONSISTENCY OF
FUNDING IN THE FUTURE.
THAT'S A HUGE PART OF GETTING
PEOPLE TO FOCUS ON AN AREA.
THE MATURITY OF THIS SPACE
INFORMS THE QUESTION.
AND WITH EXTERNAL, CONTROL.
SO, I THINK IT IS EASIER TO HAVE
A MORE HOLISTIC AGENDA THAT
LOOKS AT THE PIPELINE FROM BASIC
SCIENCE RESEARCH TO
IMPLEMENTATION WHEN SOMETHING IS
HOSTED EXTERNALLY BECAUSE YOU
CAN GET PEOPLE INVESTED IN A
DIFFERENT WAY.
YOU CAN GIVE THEM MORE
MEANINGFUL ROLES IN PART JUST
PARTLY DUE TO RULES AND RED
TAPE.
AND THAT LEADS TO A LONGER TERM
INSTITUTIONAL INVESTMENT FROM
THE PARTNERS.
BUT, THERE ARE A LOT OF
DIFFERENT PEOPLE WITH DIFFERENT
OPINIONS ON PRIORITIES AND ITS
TAKES MORE TIME AS LAURA SAID
AND THERE IS A WHOLE NORMING
PERIOD TO THE PRIORITIES THAT
OTHER STAKEHOLDERS HAVE WHEN YOU
BRING THEM IN AS EQUAL PARTNERS
MAY DIFFERENT THAN WHEN YOU
THOUGHT THEY WERE INVITED
GUESTS.
YOU HAVE TO BE READY FOR THAT
AND READY FOR THE FACT THAT
PEOPLE'S PRIORITIES MAY SHIFT
OVER TIME.
I ESPECIALLY ON THE INDUSTRY END
INVOLVING INDUSTRY, INDUSTRY ARE
A SHORTER CYCLE THAN WE ARE.
SO THEIR PRIORITIES MAY CHANGE
IN SIX MONTHS, A YEAR PRETTY
DRAMATICALLY AND DIVESTMENT UP
CERTAIN AREAS.
THE REALITY IS WE CANNOT COUNT
ON -- YOU CAN CONTRACTUALLY
OBLIGATE COMPANIES OWN A
CONTRACT FOR FIVE OR SIX-YEAR
PERIOD BUT CAN'T GUARANTEE THEIR
ORGANIZATIONAL BUSINESS
PRIORITIES WILL STAY THE
STATEMENT.
THIS COMES TO THE GENERAL IDEA
OF WHAT YOU MEAN BY HOME.
IS HOME THE INSTITUTION THAT IS
MORE DAMAGING?
AND SO I THINK THAT AS LONG AS
THERE'S COMMITMENT WITHIN THE
NIH TO OVER A LONG PERIOD OF
TIME PROVIDE THAT MONEY AND TO
HAVE CONSISTENCY OR OTHER
SOURCES OF FUNDING AND
APPROPRIATIONS, THAT'S GREAT.
THE LAST THING YOU WANT TO DO IS
GIVE SOMETHING A FOUR-YEAR TIME
LINE AND HAVE IT DISSOLVE BEFORE
ACCOMPLISHING ITS GOALS BECAUSE
WE ALL KNOW MOVING THE NOEEDLE N
THINGS LIKE THIS TAKES WORK.
HOPEFULLY THAT'S HELPFUL.
>> QUICK TECHNOLOGY QUESTION
FROM THE EXTERNAL FOLKS AND THEN
A MORE GENERAL QUESTION.
FIRST, A LOT OF LYMPHATIC
RESEARCH FUNDING IS CONGRSAL -
CONGRESSIONALLY DIRECTED AND
LOBBY EFFORTS PREVENTS A
POSITIVE FEEDBACK LOOP.
IS IT THE SAME FOR PEOPLE IN
PCORI OR THE FNIM OR IS THERE A
DIFFERENCE IN THAT SPACE.
>> IN TERMS OF LOBBY?
>> SO, ARE YOU ABLE TO
COMMUNICATE WITH CONGRESS AND
SAY, HEY, THIS IS A PRIORITY FOR
US.
WE'D LIKE THIS TO BE WRITTEN
INTO NEXT YEAR'S APPROPRIATION
OR IS THAT NOT PERMITTED FROM
WHERE YOU ARE.
>> FIRST, WE HAVE TEN-YEAR
FUNDING WHICH IS AN ADVANTAGE
AND ALSO WE DON'T LOBBY PER SE
IN THAT WAY TO BRING PRIORITIES.
OUR PRIORITIES COME FROM
STAKEHOLDERS.
SO STAKEHOLDERS WOULD BE THE
ONES TO SPEAK WITH CONGRESS
ABOUT HOW THEY FEEL PCORIs DOING
AND WHAT IS IMPORTANT FOR PCORI
TO DO IN THE FUTURE.
AND I THINK THEY MORE THAN WE
SPEAK IN THAT REGARD WITH
RESPECT.
WE TALK TO CONGRESS.
IT'S ABOUT EDUCATION AND
AWARENESS BUILDING OF THE THINGS
WE'RE CONTRIBUTING AND HOW WE'RE
CONTRIBUTING AND ARE ALWAYS
HAPPY TO TALK IF THEY HAVE
SPECIFIC QUESTIONS BUT IT'S
FEDERAL MONEY AND TRY TO RESPECT
THE RULES THAT ARE THERE
ACKNOWLEDGING SOME OF THE
DIFFERENCES AGAIN BECAUSE WE'RE
NOT REPRESENTED BY THE
ADMINISTRATIVE PORTION OF THE
FEDERAL GOVERNMENT SO THERE'S
WAYS WE TALK MORE DIRECTLY
ESPECIALLY WHEN IT COMES TIMES
WITH REGARD TO THE
APPROPRIATIONS BUT IT'S NOT
ABOUT BRINGING OUR PRIORITIES
FORWARD.
>> WHAT ABOUT THE FOUNDATION?
WITH
>> WE DO NOT LOBBY.
WE DO EDUCATION OF CONGRESS.
THEY'RE NOT THE SAME THING,
OBVIOUSLY.
WE DO NOT ATTEMPT TO DEAL WITH
LARGE CONGRESSIONAL S FOR OUR
PROGRAMS.
THEY'RE LARGELY COMING THROUGH
EXISTING PRIORITIES WITHIN NIH
OR PRIVATE SECTOR.THEY'RE LARGE
EXISTING PRIORITIES WITHIN NIH
OR PRIVATE SECTOR.
>> IT'S CHANGING PRACTICES AND
CLINICIAN EDUCATION AND IT HAS A
LOT OF DIFFERENT THINGS AND SITS
OUTSIDE OF DIRECT RESEARCH.
CAN YOU SPEAK TO WHETHER AN
INTERNAL VERSUS EXTERNAL SCIENCE
THAT WAS TALKED ABOUT, HOW DOES
THAT VIEW WITH REGARDS TO THOSE
KIND OF THINGS.
SPECIFICALLY WHAT DOESN'T WORK
WITH THE NIDDK CENTERS VERSUS
PCORI.
WHAT DOES WORK?
I WOULD LIKE TO HEAR BOTH GOOD
AND BAD.
>> MY PRIOR EXPERIENCE WAS WITH
THE NATIONAL GENOME RESEARCH
INSTITUTE WHICH IS CENTERED
FOCUS AND BASIC RESEARCH AND
ALSO TRANSITIONALLY MINDED AND
WE ALSO SPOKE TO PAYER
COMMUNITIES.
PCORI LOOKS TO ENSURE OUR PAIR
STAKEHOLDERS KNOW WHAT WE'RE
DOING AND MEETING OUR NEEDS AND
CONTRIBUTING TO FUNDING AND ONE
THING THAT IS DIFFERENT IS WE
CAN'T HAVE FULL CONVERSATIONS
WITH OTHER AGENCIES BECAUSE
WE'RE NOT PART OF THE GOVERNMENT
SO THERE'S ASPECTS TO BEING NIH
WHERE WITH A SISTER AGENCY YOU
CAN HAVE COVERINGS WITH CMS OR
OTHERS AND WE'RE HAPPY TO SPEAK
WITH THEM AND ANSWER QUESTIONS
AND WANT TO BE INFORMATIVE SO
FROM THAT PERSPECTIVE IS THE
EVIDENCE WE ARE FUNDING HELPFUL
TO YOU IN YOUR WORK BUT WON'T
HAVE THE SAME CONVERSATION WITH
THEM A SISTER AGENCY WILL HAVE.
>> THE FOUDATION IS A LITTLE BIT
IN THE MIDDLE USED TO BE WITH A
BADGE TO COME IN BECAUSE FNIMH
EMPLOYEES ARE BADGED AND WE HAVE
MORE FLEXIBILITY IN WHAT
PRIORITIES ARE.
IN A PUBLIC FORUM YOU WON'T GET
COMPANY REPRESENTATIVES TO TALK
ABOUT THEIR PRIORITIES.
WE DON'T WANT THEM TO KNOW TRULY
PROPRIETARY INFORMATION AND
THINGS TANGENTIAL TO THAT
COMPANIES WON'T SHARE IT IN
PUBLIC FORUM.
THEY'LL SHARE IT ONE-ON-ONE AND
SO WE HAVE MORE FLEXIBILITY TO
ACTIVELY ENGAGE THE
STAKEHOLDERS.
WE HAVE ABILITY TO DIRECTLY
CONTRACT AND NOT ISSUE
COMPETITIONS AND WE CAN MOVE
FASTER AND THAT'S A BIG
ADVANTAGE IN CERTAIN CASES.
WE'RE A SEPARATE NONPROFIT.
I THINK THOSE ARE THE TYPES OF
THINGS YOU'RE ASKING ABOUT.
THE DRAW BACKS ARE EVERYTHING IS
CONVINCING EVERYONE TO DO IT.
SO WE DON'T HAVE A LOT WE DON'T
FUNCTION FROM ENDOWMENT.
WE HAVE TO FUND RAISE FOR OUR
PROGRAMS AND FOLLOW OTHER
PEOPLE'S PRIORITIES.
IT'S THE ART AS I MENTIONED OF
BUILDING CONSENSUS AND ALIGNING
STAKEHOLDERS AND THAT'S HARD
WHEN YOU DON'T HAVE A BASE
AMOUNT OF FUNDING TO START FROM.
IN THIS CASE IF YOU LOOK AT
APPROPRIATIONS OR SOMETHING
SIMILAR, THAT MAY NOT BE THE
CASE AND THAT'S NOT THE CASE FOR
PCORI OPERATING FROM AN EXISTING
BASE.
>> I WOULD LOVE TO INVITE THE
COPD TEAM TO ALSO OPINE ON THE
QUESTION TO GIVE ANOTHER
PERSPECTIVE.
>> A LOT OF THINGS TOUCHED THE
COPD AREA IN THERE'S NO
SET-ASIDES AND THE CONGRESSIONAL
MANDATE CAME WITH PUT THE PLAN
TOGETHER BUT DIDN'T COME WITH
FUNDING TO DO ANYTHING.
THERE'S NOTHING IN THERE THAT
SAYS CREATE THIS OR THAT.
IT'S MEANT TO BRING TOGETHER THE
COMMUNITY TO USE WHATEVER
RESOURCES ARE AT THEIR DISPOSAL
TO MAKE IT A PRIORITY.
CLEARLY FOR NHLBI THIS IS A
PRIMARY AREA FOR LUNG PROGRAM.
IT'S A BIG AREA FROM
IMPLEMENTATION AND OUTREACH AND
EDUCATION.
AND IT'S BEEN THAT WAY FOR A
WHILE SO IT ISN'T ANYTHING NEW
FOR US.
IN A WAY THIS IS AN ATTEMPT TO
GO AFTER THE BIGGEST POOL OF
RESOURCE OUT THERE THE RO1 POOL.
FOR US WHAT WE'VE DONE IS TRIED
HARD USING A TRACKING TOOL AND
OTHER MEANS TO PUSH THE
COMMUNITY INTO THAT POOL AND SAY
YOU DON'T NEED US TO TELL YOU TO
CREATE CENTERS.
YOU DON'T NEED US TO TELL YOU TO
DO TRIALS.
WE DON'T NEED TO TELL YOU WHAT
MECHANISM TO USE BECAUSE EVERY
MECHANISM IS AVAILABLE TO YOU.
COME TO US AND BRING US YOUR
BEST IDEAS AND HOW YOU WANT TO
CONSTRUCT THEM AND WE'LL TELL
YOU WHETHER THAT'S SOMETHING WE
CAN EMBARK ON OR CONSIDER AND I
THINK THAT'S HOW WE'VE BEEN
APPROACHING THIS AND AGAIN IT'S
A WORK IN PROGRESS SO WE DON'T
HAVE THE ANSWER TO THIS BUT HAVE
USED A VARIETY OF MECHANISM TO
STIMULATE THE FIELD AND OTHER
AREAS WE DON'T TOUCH ON QUITE AS
EXTENSIVELY BUT OUR PARTNERSHIPS
ARE PRETTY BROAD.
WITH OUR EFFORTS WITH CDC THEY
HAVE A DEDICATED AREA COLLECTING
PREVALENCE INCIDENT DATA AND
TRACKING MORTALITY AND HEALTH
CARE UTILIZATION.
THERE'S DIFFERENT WAYS AND
YOU'RE HEARING THEM THROUGH THE
FOUNDATION AND KIDNEY CENTERS
AND OTHER WAYS AND I THINK AGAIN
SOMEBODY SAID IT COMES DOWN TO
THE QUESTION THAT YOU'RE TRYING
TO ADDRESS WHERE THE FIELD IS
AND MOST POTENTIAL IS FOR NOT
ONLY A STREAM OF SUPPORT TO
ALLOW YOU TO CAPITALIZE ON
POTENTIAL OPPORTUNITIES BUT AT
THE SAME TIME THE SUSTAINABILITY
AND HOW TO MAINTAIN THAT OVER
THE YEARS AND THAT'S IMPORTANT
BECAUSE YOU DON'T WANT THIS TO
JUST BE A BLIP ON THE RADAR
SCREEN AND THEN GONE.
IT'S SCIENCE THAT NEEDS TO
CONTINUE ON AND ON.
I TRIED TO GIVE A PERSPECTIVE A
LITTLE BIT FROM WHERE WE ARE
WITH THE COPD AREA BUT I THINK
IT HAS RELEVANCE TO ALL THE
POINTS AND QUESTIONS THAT WERE
ASKED PREVIOUSLY TO THE OTHER
SPEAKERS AND PANELISTS.
>> I WANT DO MAKE SURE ALL OUR
COMMITTEES HAVE IN AEQUITABLE
OPPORTUNITIES TO ASK QUESTIONS.
I'LL TURN IT OVER TO THE PATIENT
TOOL COMMITTEE.
>> THE COMMITTEE HAS DISCUSSED
WAYS TO QUANTIFY THE PEOPLE IN
THE LYMPHATIC DISEASE.
HOW DID YOU GET TO THE 17
MILLION DIAGNOSED.
ICD10 CODES OR OTHER MEANS OF
GETTING TO THAT NUMBER?
>> THE SHORT ANSWER IS THAT
THESE NUMBERS ARE DEVELOPED BY
CDC.
THEY USE THEIR OWN METHODOLOGY
AND TRACKING SYSTEM.
THEY HAVE A COMPLETELY
SOPHISTICATED WAY TO REPORT ALL
DISEASE ENTITIES AND PRODUCE
THAT AND PUT OUT PUBLICATIONS
WITH MORTALITY AND MORBIDITY
DATA.
WE RELY ENTIRELY ON CDC TO MAKE
SURE THE ESTIMATES ARE
APPROPRIATE AND REPRESENTATIVE
ON THE NATIONAL SCALE.
IS THAT ABOUT RIGHT NOW?
>> THAT'S CORRECT.
SOMETIMES WE BREAK IT DOWN A
LITTLE BIT FURTHER.
WE HAVE PREVALENCE DATA BUT IT'S
ALSO FROM THE BEHAVIORAL RISK
FACTOR AND CDC PUT OUT AND THAT
WAY WE CAN MESSAGE AND X NUMBER
OF PEOPLE IN YOUR STATE HAVE
COPD.
WE ENCOURAGE PEOPLE THEN IN OUR
PARTNER NETWORKS TO TAKE THAT TO
LOCAL LEGISLATORS AND MEDIA TO
HELP BUILD AWARENESS AROUND WHAT
THE PREVALENCE OF THE DISEASE IS
IN THEIR STATE AND WHY PEOPLE
SHOULD PAY ATTENTION.
IT'S ALL CDC'S DATA BECAUSE
THAT'S THEIR ROLE.
>> THANK YOU.
THE NEXT QUESTION IS FOR ALL THE
STAKEHOLDERS.
HOW DO YOU DECIDE WHAT
INFORMATION TO DISSEMINATE TO
PATIENTS AND WHAT HAVE YOU FUND
TO BE THE BEST MEDIUM TO DO
THAT?
WHAT HAS BEEN MOST EFFECTIVE
MEDIUM TO USE?
>> I DON'T KNOW IF YOU'LL WANT
TO HEAR FROM THE COPD SIDE BUT
WE HAVE LOOKED INTO THIS
EXTENSIVELY.
MAYBE TOUCH ON HOW WE NOT ONLY
CAPTURED WHAT HEALTH EDUCATION
MATERIALS GO OUT BUT HOW WE
DISTRIBUTE THROUGHOUT THE
NETWORK WE CREATED WITHIN NHLBI
TO DISSEMINATE THAT INFORMATION?
YOU MAY WANT TO QUICKLY LET THEM
KNOW HOW THAT IS WORK.
>> WE HAVE A NATIONAL LUNG
HEALTH EDUCATION PROGRAM WE USE
FOR DIFFERENT COMMUNITIES.
WE HAVE A LARGE OUTREACH ARM AND
EDUCATION WE'RE DOING AND IF
YOU'RE ASKING THE BEST WAY, I
CAN'T SAY THERE IS ONE BEST WAY.
WE ALWAYS UTILIZE THE SURROUND
SOUND APPROACH.
IT DEPENDS ON THE COMMUNITY
YOU'RE TRYING TO REACH.
THE FACT SHEET AND SOMETIMES
PEOPLE WANT THEM IN DOCTOR'S
OFFICE OR RADIO ADS, SOCIAL
MEDIA, WEBINARS.
I DON'T THINK THERE'S A ONE SIZE
FITS ALL APPROACH.
IT'S THE GOAL OF WHAT YOU'RE
TRYING TO DO WILL DICTATE HOW
YOU DO IT AND WHO TO GET
INVOLVED AND WE HAVE OUR BRIEF
BETTER NETWORK A PARTNERSHIP
AROUND THE COUNTRY WE COORDINATE
TO HELP FACILITATE DIALOGUE
ABOUT COPD AND LUNG CONDITIONS
AND TRY TO FIND OUT FROM
PARTNERS THE RESOURCES YOU'RE
CREATING AND KEY PUBLIC HEALTH
ISSUES AND WHAT ARE YOU DOING.
LET US KNOW TO SHARE THAT WITH
THE PARTNERS TRYING TO AVOID
DUPLICATION OF EFFORTS AND
SHARING OF INFORMATION AND
RESOURCES.
WE TALKED TO OUR PARTNERS
REGULARLY AND HAVE A LIST SERVE
AND ON SOCIAL MEDIA AND
EVERYTHING.
IT INCLUDES DIALING THAT NEEDS
TO HAPPEN AND MAKE SURE YOU HAVE
THE CONVERSATIONS FREQUENTLY TO
FIGURE OUT THE PRIORITIES AND
WHAT'S THE INFORMATION THAT'S
NEEDED.
>> QUICKLY TO ECHO AND REINFORCE
EMPHATICALLY THERE'S NO ONE
RIGHT WAY AND IT'S AUDIENCE
DEPENDENT.
ONE OF THE THINGS THE COLLECTION
OF THINGS PCORI HAS DONE IS
RECEIVE ACTIVITIES AND TO PUT
FUNDING AS WELL INTO THOSE SO IT
IS YES, THERE'S DISSEMINATION
AND PROMOTION OF AWARENESS WE
CAN DO BUT PARTNERING WITH
COMMUNITY ORGANIZATIONS TO BUILD
DISSEMINATION TOOLS AND UTILIZE
THOSE NETWORKS THAT THEY HAVE TO
GET FINDINGS OUT TO THEIR
ORGANIZATIONS.
WE HAVE THE ENGAGEMENT AWARDS TO
PROVIDE FUNDS FOR DIFFERENT
COMMUNITIES TO DO DISSEMINATION
AND BUILD TOOLS THAT ARE THEN
AVAILABLE TO THE ORGANIZATIONS,
ETCETERA.
AND I THINK I'LL HIT ON AGAIN
THERE ARE THINGS WE NEED TO
LEARN ABOUT HOW TO DO THIS
BETTER AND SO IT'S THE PRACTICE
AND THEN ALSO THE LEARNING THAT
NEEDS TO GO ON.
WE HAVE SOME MORE CER RELATED
ACTIVITIES AROUND SCIENCE OF
ENGAGEMENT AND AROUND
COMMUNICATION IN TERMS OF
UNDERSTANDING WHAT'SING ABOUT TO
BE MEANINGFUL AND HOW TO
COMMUNICATE WITH DIFFERENT
AUDIENCES AND PARTNER WITH
DIFFERENT AUDIENCES AND AGAIN
CAPACITY THROUGH HSII LEARNING
HOW TO SCALE AND GET THIS OUT
AND WORKING WITH THE PARTNERS AT
THE TABLE FROM CONCEPT TO
IMPLEMENTATION TO UNDERSTAND
WHAT THEY NEED UP FRONT SO IT'S
BUILT AND THEN TO MAKE SURE IT'S
SUCCESSFULLY TRANSLATED OUT TO
THE RIGHT GROUPS.
>> I WANT TO REITERATE -- YOU
MADE A FEW IMPORTANT POINTS.
ONE IS DON'T FINISH A PROJECT
AND THEN GO TO PEOPLE AND ASK
THEM TO DISSEMINATE THE RESULTS.
INVOLVE THEM FROM THE BEGINNING
BECAUSE THEN THEY'LL BE INVESTED
AND COMMUNICATE AS THEY'RE
RELEVANT TO THEIR COMMUNITIES.
SECOND IS BUILD ON OTHER
PEOPLE'S PLATFORMS.
DON'T TRY TO CREATE A NEW
PLATFORM YOURSELF.
THAT MEANS ARE YOU GOING TO
DEVELOP THE MOST WATCHED TIKTOK
OR INSTAGRAM CHANNEL THAT
EXISTS?
NO, PROBABLY NOT.
UTILIZE EXISTING NETWORKS THAT
MIGHT CARE ABOUT WHAT YOU'RE
DOING.
AND THIS IS A SEPARATE ONE FROM
MY OLD DAYS FROM MY PATIENT
ADVOCACY HAT IS ABOUT THE EGO
AROUND PROJECTS AND PROGRAMS.
WHEN PEOPLE HAVE -- AND WE RUN
INTO THIS PROBLEM ALL THE TIME.
WHEN YOU HAVE AN AMAZING
FINDINGS ALL YOU WANT TO TALK
ABOUT IS THAT FINDING.
THAT'S NOT WHAT PATIENTS CARE
ABOUT.
THEY CARE ABOUT THAT FINDING IN
THE CONTEXT OF THEIR CONDITION.
AND SO YOUR AMAZING FINDING
MIGHT BE LIKE FIVE MINUTES OF A
30 MINUTE PODCAST AND THAT
THOUGH IS WHERE PEOPLE ARE GOING
TO CARE IF IT'S PUT IN THE
CONTEXT OF THE INFORMATION THEY
CARE ABOUT AS A WHOLE.
THEY WILL LISTEN AND HEAR IT AND
USE IT.
BUT IF YOU DO A 30-MINUTE
PODCAST ON THE SCIENTIFIC
FINDING, PATIENT, COMMUNITIES,
WON'T TUNE INTO THAT.
SO THERE IS AN EGO COMPONENT TO
TAKE A STEP BACK AND SAY, HOW DO
YOU NOT PRESENT THE INFORMATION
HOW YOU WISH IT WAS PRESENTED
WITHIN POINTS YOU WISH IT WAS
AND HOW HOW DO YOU LOOK AT THE
AUDIE
AUDIENCE.
>> WE HAVE A COMMUNITY
SUBCONTRACT AND IT'S NOT A LOT
OF MONEY BUT OFFER TO NON-PROFIT
ORGANIZATIONS TO DO INNOVATIVE
HEALTH EDUCATION PROJECTS AND
MAP BACK TO TALK ABOUT HOW IT
FURTHERS IMPLEMENTATION OR A
NEEDS IN THEIR COMMUNITY AND ARE
PILOT PROGRAMS OF WHAT WORKS IN
THAT COMMUNITY AND ONE CASE
WE'VE BEEN ABLE TO TAKE IT ON A
NATIONAL LEVEL WAS A CAREGIVER'S
TOOL KIT OUR ORGANIZATION
STARTED TO CREATE AND THEN WE
SAW GREAT RESULTED AND NOW IT'S
OFFERED AS A GOOD JOINT RESOURCE
NATIONALLY AVAILABLE.
IT'S NOT A LOT OF MONEY BUT
COMMUNITY ORGANIZATIONS CAN DO A
LOT AND YOU LEARN A LOT ABOUT
YOUR COMMUNITIES IN DOING THOSE
THINGS.
>> WE WERE TALKING ABOUT
REACHING OUT TO COMMUNITIES NOT
AS EASILY REACHED OR ENGAGED.
HOW ARE ORGANIZATIONS
IDENTIFYING A SUCCESSFUL
OUTREACH?
DO WE NEED TO EXPAND ON THAT IN
PARTICULAR OR ON THE REVERSE
SIDE IF THERE WERE IDENTIFIERS,
HOW DO YOU MEASURE THAT?
>> I THINK IT'S AN AREA WHERE WE
NEED METRICS AND METRIC
DEVELOPMENT.
THINKING ABOUT THIS THE WORK TO
BE DONE AND THE SCIENTIFIC
ENGAGEMENT PROGRAM PART OF OUR
METHODS TO SUPPORT CER IS ABOUT
THAT IN TERMS OF ENGAGING FOLKS
AND THAT INCLUDES DISSEMINATION
ACTIVITY AND UNDERSTANDING
DIFFERENT COMMUNITY NEEDS AND
HOW TO PUT TOGETHER MEANINGFUL
PARTNERSHIPS WITH THOSE
COMMUNITIES.
IT'S AN AREA WE NEED TO CONTINUE
WORKING ON AND WE FOCUS AGAIN
MORE SO AT THE HIGHER LEVEL
TRYING TO LOOK FOR WHAT SIGNALS
ARE THERE AT THE MOMENT WHICH
ARE MOVING THINGS INTO PRACTICE
AND SMALLER ACTIVITIES THAT WE
HAVE THAT CAN GET CLOSER TO THE
GROUND AND THINK ONE OF THE
THINGS PCORI TAKES SERIOUSLY IS
LOOKING ACROSS THE PORTFOLIO AS
WE HAVE FUNDED PROJECTS LOOKING
AT DIFFERENT SPACES WHAT ARE
RELEARNING ACROSS THE BOARD AND
HOW CAN WE GET THAT OUT AS WELL
WHETHER TO THE RESEARCH
COMMUNITY, HEALTH COMMUNITY OR
DIFFERENT COMMUNITY PARTNERS SO
THAT IT DOESN'T STAYED BURIED
WITHIN APPROVALS AND SILOED AS
SUCCESS STORIES BUT HIGHLIGHTED
IT.
>> LOOKING AT WHAT PUBLIC HEALTH
HAS DONE FROM OUTREACH AND
MEASUREMENT AND MEASURE IMPACT
AROUND PRODUCTS AND SALES AND
RETAIL AND THOSE TYPES OF
THINGS.
A LOT OF THE MEASURES THEY USE
INTERNALLY ARE TRIED AND TRUE
METRICS.
SO USING THOSE BECAUSE THE WORK
HAS ALREADY BEEN DONE IS
INCREDIBLY HELPFUL.
>> WE'LL GO TO ALISSA AND
GUILLERMO AND THEN FROM THE
IMPERATIVE COMMITTEE.
>> THE PATIENT TOOL COMMITTEE IS
INSPIRED BY ACTION.
COULD THE COPD TEAM SPEAK TO
WHAT IT MAY TAKE TO FORM AN
LYMPHATIC ACTION PLAN AND DO YOU
RECOMMEND WE HAVE ONE.
>> I'LL TAKE THE FIRST PASS AND
NEYAL MAY HAVE MORE TO OFFER.
THE SHORT ANSWER IS THIS IS A
VERY IMPORTANT EFFORT IF YOU
WANT TO TRY TO GALVANIZE THE
COMMUNITY OR TRY TO GET THE
STAKEHOLDER INPUT OR PUT
TOGETHER WHAT MIGHT BE CALLED A
BLUEPRINT ROAD MAP, WHATEVER
METAPHOR YOU WANT TO USE BUT YOU
NEED TO START SOMEWHERE TO BRING
IT ALL TOGETHER IN A WAY THAT
THE COMMUNITY BUYS INTO IT AND
THEN YOU FIGURE OUT HOW TO
IMPLEMENT THAT.
AND I THINK THIS WAS VERY
IMPORTANT WAY IN WHICH WE WERE
ABLE TO SAY WHERE ARE WE IN THE
DIFFERENT CATEGORIES AND WHERE
DO WE NEED TO GO.
AND THEN HOW DO WE GET THERE.
IT IS A PROCESS.
IT'S A STEP WISE PROGRESSION BUT
I THINK IT'S AN IMPORTANT STEP
OR YOU'RE GOING TO HAVE LOTS OF
THINGS FLYING IN THE AREA AND
HOW ARE YOU GOING TO PRIORITIZE,
HOW ARE YOU GOING TO DETERMINE
WHERE YOU'RE GOING TO FOCUS
RESOURCES IF THAT'S AVAILABLE TO
YOU AND HOW ARE YOU GOING TO
MAKE THE MOST CHANGE YOU'RE
LOOKING FOR.
IT'S ALL ABOUT IMPACT.
HOW CAN YOU PUT SOME RESOURCES
TOWARDS THINGS THAT WILL GIVE
YOU THE GREATEST IMPACT AT LEAST
WHAT YOU MIGHT THINK MIGHT BE AT
THIS POINT.
IN MY VIEW I THINK IT'S
CRITICALLY IMPORTANT FIRST STEP
IN BRINGING IT ALL TOGETHER.
I DON'T KNOW IF YOU WANT TO ADD
MORE TO THAT, NEYAL.
>> THINKING WHERE THE HOME
SHOULD BE IT'S A TOUGH QUESTION
BECAUSE SOMETIMES PART OF ME
WISHES THE HOME WAS SOMEWHERE
ELSE AND WE DON'T OWN IT BUT
HELPING FACILITATE AND MORE IT
FORWARD AND A LOT OF IT FALLS ON
NHLBI'S SHOULDERS TO KEEP IT OUT
IN THE COMMUNITY AND TOP OF
MIND.
IT'S A LOT OF WORK.
THERE'S THINGS WE CAN DO AS A
GOVERNMENT AGENCY AND CAN'T DO
THAT MAY DRIVE THINGS FORWARD.
THINK ABOUT THE ORGANIZATION
PULLING IN AND WHAT'S IN IT FOR
THEM AND WHY SHOULD THEY CARE
AND HOW TO KEEP IT MOVING
FORWARD AND IS THIS COMMISSION
GOING TO SPEARHEAD THAT AND MOVE
IT ALONG BECAUSE IT NEEDS
SOMEONE TO SHEPHERD THE EFFORTS.
IT TAKES A LOT.
>> THANK YOU SO MUCH.
FOLLOW-UP QUESTION.
IF YOU WERE TO DO IT AGAIN, HOW
WOULD YOU DO IT DIFFERENTLY THIS
TIME AROUND?
>> WELL, ONE OF THE THINGS I
THINK YOU'VE HEARD OVER AND OVER
AGAIN IS THAT IT'S GREAT TO HAVE
A PLAN BUT YOU ALSO NEED
RESOURCES TO IMPLEMENT THE PLAN.
SO, IT WOULD BE NICE IF WHETHER
IT COMES THROUGH OUR OWNER
APPROPRIATIONS PROCESS OR
WHETHER THERE'S DEDICATED
RESOURCES COMING IN FROM
INDUSTRY OR THE PRIVATE SECTOR
OR WHEREVER IT'S KIND OF WHERE
THE FNIH CAN HELP THE EFFORT AS
WELL BUT AGAIN I THINK WE DIDN'T
GO OUT THERE SAYING WE WANTED
$20 BILLION TO DO THIS.
WE DIDN'T GO WITH A BIG ASK.
WE SAID LET'S TAKE A LOOK AT
WHERE THE STATE OF THE FIELD IS,
WHERE DO WE NEED TO GO AND SEE
IF WE CAN CHIP AWAY AT IT USING
OUR EXISTING APPROACH.
I THINK BY AND LARGE I WOULDN'T
DO MUCH DIFFERENT IF I HAD TO DO
IT OVER AGAIN OTHER THAN TO SAY
I WOULD HAVE LIKED A REVENUE
STREAM THAT WOULD HAVE BEEN ABLE
TO SUSTAIN IT BEYOND JUST TRYING
TO UTILIZE WHAT COMES TO US
THROUGH OUR NORMATIVE PROCESSES
BECAUSE IT'S COMPETITIVE AND
THERE'S ONLY SO MUCH WE CAN DO
AND YOU REALLY COULD USE A
LITTLE BIT OF HELP TO REALLY
SHORE IT UP.
MAYBE THAT WOULD BE THE ONE
PIECE I WOULD HAVE ADDED.
I DON'T KNOW NEYAL IF YOU THINK
THERE'S OTHER THINGS THAT WOULD
HAVE BEEN DONE DIFFERENT?
>> LARGELY, THERE'S NOT TOO MANY
THINGS DIFFERENTLY.
I THINK THE REVENUE IS IMPORTANT
BECAUSE I THINK ABOUT THAT
SUBCONTRACT PROGRAM I MENTIONED.
IT'S A SMALL AMOUNT AND ONLY
ABOUT $150,000 A YEAR WHICH IS
NOT VERY MUCH MONEY BUT HAD WE
HAD MORE TO DO COMMUNITY-BASED
INTERVENTIONS I THINK THAT WOULD
BE FANTASTIC.
I THINK THE OTHER PIECE TO DO
OVER AGAIN IS TRY TO FIGURE OUT
HOW TO HOLD SOME OF THE PARTNERS
AND STAKEHOLDERS MORE
ACCOUNTABLE IN TERMS OF TAKING
OWNERSHIP OVER THE GOALS OR
OBJECTIVES AND MORE REGULAR
REPORTING OR MEETINGS AND WHY
SHOULD AN AGENCY OR SOMEONE
FACILITATE A CERTAIN GOAL AREA
BUT I THINK THAT IS A CHALLENGE
FOR US TO GO AFTER THE POLICY
AND WE DON'T REALLY DO POLICY OR
TO THE OTHER PUBLIC HEALTH AREAS
THAT ARE NOT NECESSARILY UNDER
OUR PORTFOLIO OR WHAT NHLBI
SHOULD BE DOING THEM BUT KEEP
AFTER THEM AND GET ORGANIZATIONS
TO BE ACCOUNTABLE TO THE ACTION
PLAN IF YOU DEVELOP ONE THAT'S
AN IMPORTANT ELEMENT AND REPORT
OUT ON THEIR OWN PROGRESS TO THE
LARGER COMMUNITY.
>> AND NO MATTER WHERE THE HOME
IS IF YOU ALLOW PEOPLE TO
SELF-ORGANIZE WITHIN WITH AN
OVER ARCHING PLAN AND WITH
SHARED ACCOUNTABILITY,
WONDERFUL.
IT WASN'T A MATTER OF FINDING A
HOME WHICH IS A LITTLE DIFFERENT
FROM WHAT YOU'RE ADDRESSING
RIGHT NOW WHICH IS WHERE THE
HOME MIGHT BE IF THERE WAS SUCH
A THING AND WHAT WOULD BE THE
MOST ADVANTAGEOUS PLACE TO PUT
IT.
THIS WAS AGAIN TO SET UP OKAY,
NOW WE KNOW WHERE THE HOME IS
BUT WE NEED MORE THAN THE HOME
TO HAVE THE IMPACT WE'RE LOOKING
FOR AND THEN SET THE GOALPOST
AND THEN GO FROM THERE I THINK
WE'RE A LITTLE BIT DIFFERENT
FROM SOME OF THE THINGS YOU'RE
ADDRESSING WHICH ARE ALL VERY
IMPORTANT TO THINK ABOUT UP
FRONT BUT MAYBE IN A DIFFERENT
PLACE WHEN IT COMES TO THE HOME
QUESTION.
>> IN THE FEEDBACK YOU HAVE THIS
BUT DIFFERENT TO THE KIDNEY
PEOPLE ARE FAMILIAR WITH AND
THERE'S AN ADDITIONAL STEP TO
EDUCATE PEOPLE WHOEVER WE'RE
TALKING, DONORS, PRIVATE, BIO
TECH, ETCETERA.
THIS IS A MAJOR CHALLENGE
BECAUSE LEARNING IN FIVE MINUTES
ABOUT LYMPHATICS.
MOST PEOPLE ARE NOT FAMILIAR
WITH THE TOPIC AND SOMETHING WE
NEED TO REMIND OURSELVES
WHATEVER WE'RE GOING TO DO WE
NEED TO INCLUDE THAT ASPECT
BECAUSE OTHERWISE YOU WILL NOT
BE ABLE TO SUCCEED IN CONVINCING
PEOPLE THIS IS A RELEVANT
ASPECT.
THERE'S A SMALL NUMBER OF BIO
TECH.
WE NEED TO RESPECT THE POINT OF
VIEW DIFFERENT FROM THE KIDNEY
AND LUNG AND EVERYBODY WITH THE
DISEASES AND DISORDERS.
I THINK THAT'S SOMETHING I WOULD
LOVE TO HEAR ANY OF YOU OF THE
OTHER GROUPS TO GIVE US SOME
ADVICE HOW TO GO AROUND AND TRY
TO EDUCATE AT EVERY DIFFERENT
LEVEL.
WHATEVER WE WANT TO ACCOMPLISH
WE COULD DO IT BECAUSE WE DON'T
NEED TO GO THROUGH THE EXTRA
STEPS EVERY TIME WE'RE GOING TO
TRY TO DO SOMETHING.
>> I CAN START A LITTLE BIT
THERE.
YOU'D BE SURPRISED HOW FEW
PEOPLE KNOW WHAT COPD IS.
>> THEY KNOW IT'S THE LUNG.
>> ONE THING WE DID WHEN I
STARTED AT NHLBI WE DID NATIONAL
SURVEYS OF THE HEALTH CARE
PROVIDER COMMUNITY AND GENERAL
PUBLIC 18 AND OLDER TO
ACKNOWLEDGE ATTITUDES AND
PRACTICE AND CAN THEY RECOGNIZE
SYMPTOMS AND ASKING QUESTIONS
AND USED THAT DATA IN TERMS OF
WHERE TO PUT ENERGY AND
RESOURCES AND WHAT ARE NEEDS OF
DIFFERENT POPULATIONS AND FACED
KNOWLEDGE GAPS AND WHAT WE TRIED
TO ADDRESS.
>> IT'S IMPORTANT BECAUSE NO
MATTER WHAT YOU THINK A DISEASE
THAT KILLS THAT MANY PEOPLE
EVERYBODY WOULD BE AWARE AND WE
WERE SHOCKED TO SEE HOW LOW THE
AWARENESS LEVELS WAS.
WE'VE BEEN CHIPPING AWAY FOR A
WHILE AND WE'RE GETTING THERE.
THIS IS SOMETHING OUR INDUSTRY
PARTNERS HAVE ALSO HELPED WITH
THOUGH FROM A DIFFERENT
PERSPECTIVE DEVELOPING
MEDICATIONS AND SUCH AND THAT'S
IMPORTANT AND THEY ARE RAISING
AWARENESS BUT IN THE LYMPHATICS
AREA YOU DO HAVE A PRETTY STEEP
HILL TO CLIMB.
IT'S PROBABLY GOING TO BE A
TOUGH ONE AND NEED TO LOOK AT
VARIES WAYS IN WHICH GROUPS HAVE
DONE THIS WHETHER WELL KNOWN
HEART, LUNG AND KIDNEY OR ONES
MORE ORPHAN OR RARE AND SEE HOW
ONE MIGHT RAISE AWARENESS.
CYSTIC FIBROSIS IS ANOTHER AREA
WITHIN OUR PURVIEW.
THEY'VE DONE A MAGNIFICENT JOB
RAISING AWARENESS.
IT'S NOT LYMPHEDEMA.
YOU'RE TALKING MORE BROADLY.
YOU HAVE A CHALLENGE TO MAKE
SURE YOU DO A PUBLIC AWARENESS
EFFORT TO BRING IT MORE TO THE
PUBLIC IN TERMS OF THE
IMPORTANCE OF LYMPHATICS AND
BEYOND THE THING THEY MAY BE
FAMILIAR WITH AND MAYBE NOT BE
THAT FAMILIAR WITH LIMB FA DE
DEMOO -- LYMPHEDEMA AND GETS
ADDRESSED YEAR AFTER YEAR.
IT WILL NEVER GET TO THE POINT
WHERE EVERYBODY WILL HAVE IT
FALL OFF THEIR TONGUE LIKE THE
OTHER THINGS THAT EVERYBODY
KNOWS ABOUT EVERY DAY.
>> I COME FROM A GENETIC AND
RARE DISEASE WORLD FOR MOST OF
MY CAREER.
IT'S WORK WITH THE PATIENT
COMMUNITIES AN UMBRELLA APPROACH
WHERE POSSIBLE IS GREAT.
IT'S IDENTIFYING FOUNDATIONS WHO
MIGHT HAVE AN INTEREST IN THE
AREA AND PROVIDE SUPPORT FOR
THIS TYPE OF WORK LAURA IS
TALKING ABOUT.
SO FREQUENTLY SCIENCE HAS
FUNDING AND EVERYTHING ELSE IS
AN EXPECTATION OF PEOPLE DOING
IT OUT OF THE GOODNESS OF THEIR
HEARTS AND FREE TIME AND THE
REALITY IS COMPANIES ARE PUTTING
THE MOST MONEY INTO MARKETING
AND EDUCATION BECAUSE THEY KNOW
THAT'S WHAT DRIVES SALES OF
THEIR PRODUCTS.
THAT'S SOMETHING WE DO NOT DO AT
ALL.
SO FREQUENTLY THERE'S NO MONEY
THERE.
MY FELLOW PANELISTS GIVE LITTLE
INFORMATION TOE ACQUIRE BECAUSE
THEY ALL UNDERSTAND THAT AND
INVEST INTO THOSE ACTIVITIES
BUDS -- BUT IS IMPORTANT TO
THINK ABOUT.
>> I HEARD A LOT ABOUT THE
COLLABORATIVE CENTERS AND I HAD
TWO QUESTIONS IN TALKING ABOUT
SHARING RESOURCES AND WHAT
RESOURCES ARE SHARED AND IF
THERE'S A DATA REPOSITORY YOU
CREATED.
WHAT DOES IT LOOK LIKE?
>> IT CAN REACH OUTSIDE THE
INDIVIDUAL CENTER BUT NATIONALLY
THAT WOULD BE A DIFFERENT
APPROACH BUT FROM A RARE DISEASE
AND PROBABLY DISPERSED DISEASE
YOU MAY CONSIDER THAT.
>> WITH SOME OF THE OTHER TO
YOUR POINT ABOUT LARGE DATA
NETWORKS WITH INFORMATION FOR
RARE DISEASE AGAIN ARE THE
PLACES TO GO TO UTILIZE FOR THE
RESEARCH THAT THEN COULD BE
FUNDED FROM THE SPECIFIC
LYMPHATICS PERSPECTIVE.
IT MAY NOT BE ALL ABOUT
CREATION.
THERE'S THINGS SPECIFIC THAT
NEED TO BE DONE AND RESOURCES TO
BEAR BUT I WANTED TO REMIND YOU
TO LOOK AT WHAT ELSE IS OUT
THERE AND ARE THERE WAYS TO
LEVERAGE TO GET THINGS STARTED.
>> LET'S SAY WE WERE BUILDING A
CENTER.
DO YOU THINK WE NEED, WE HAVE TO
IS IT OPTIONAL TO GET INDUSTRY
INVOLVED AND HOW DO YOU
INCENTIVIZE INDUSTRY TO WORK
WITH US?
>> IT DEPENDS ON WHERE YOU'RE AT
IN THE PROCESS.
COMPANIES ARE NOT GOING TO
INVEST TIME UNLESS THEY INTEREST
FINANCIAL INTERESTS SO BUT
SOMETIMES YOU'D BE SURPRISED
WHERE THEIR INTEREST LIE.
WE'VE DONE STAKEHOLDER MAPPING
AND INTERVIEWS AND SURVEYS
AROUND INFORMATION AND UNITY
PORTFOLIO AND WHERE WE WERE
SEEKING PITCHES AND PRIORITIZED
PITCHES AND GOT TWO ON GOUT.
THAT WAS NOT WHAT I WAS
EXPECTING TO GET PITCHES ON.
WE HAVE A PROJECT IN
FUND-RAISING FOR GOUT AND I
THINK IT'S GOING TO BE FUNDED.
IT IS IMPORTANT NOT TO ASSUME
AND TO REACH OUT AND FIND OUT
WHERE THERE'S INTEREST.
AND FOR THE MOST PART INDUSTRY
WILL BE INTERESTED IN EITHER
SOMETHING THAT WILL CHANGE THE
LANDSCAPE FOR INVESTMENT IN THAT
FIELD BY GENERATING LARGE
AMOUNTS OF DATA THEY WOULD FIND
USEFUL BY DISEASE OF INTEREST OR
RELATED DISEASE.
SOMETIMES YOU CAN MAKE THAT
ARGUMENT UNDERSTANDING OF A
MECHANISM OF ACTION FOR ONE
DISEASE AREA THAT WILL BE
RELEVANT TO OTHER DISEASES AS
WEL
WELL.
OR A NARROW PROJECT TO BUILD IN
SMALLER PROJECTS OF INTEREST.
INVOLVING COMPANIES AND WE WANT
DRUGS FOR MANY OF THESE DISEASES
AND TREATMENTS AND YOU MAY NOT
START THOUGH BY GETTING INTEREST
AND THAT'S OKAY.
AS WE BUILD FOUNDATIONAL WORK IN
THAT AREA THAT'S PART IN THE
RARE DISEASE SPACE WHERE YOU
HAVE TO GO IN THE INTERESTINGAL
PIPELINE AND THERE'S A ROAD MAP
OF STEPS TO TRY TO ATTRACT
INVESTMENT AND INTEREST IN
REGISTRIES AND FUNDING OF JUNIOR
RESEARCHERS AND ALL THIS STUFF.
THAT'S A BIG ANSWER AND I
REALIZE IT'S A DIFFERENT FOR
DIFFERENT DIVISIONS.
>> I DON'T WORK WITH THE
INDUSTRY IN THE SAME WAY OR HAVE
AS MUCH EXPOSURE BUT I WOULD SAY
AGAIN MY EXPERIENCE WOULD BE IT
IT'S NOT REQUIRED AND MAY BE
STEP WISE IN TERMS OF A
DIFFERENT POINTS AND DIFFERENT
SIZE PROJECTS AND PARTNERSHIPS
MAY BE RELEVANT EARLY VERSUS
WHERE WE'D HOPE TO GET TO TO THE
POINT WE HOPE TO GET TO WHERE
IT'S CLOSER TO SOMETHING IN
THEIR FINANCIAL INTEREST AREA.
SO MAYBE DON'T THINK ABOUT THAT
LONG-TERM GOAL UP FRONT BUT WHAT
ARE OTHER WAYS TO PARTNER EARLY
THAT MAY HAVE INTEREST IN
BUILDING PARTNERSHIPS AND MAYBE
IT'S FOUNDATIONS AS MUCH
INDUSTRY IN THE BEGINNING.
>> WE'RE QUICKLY RUNNING OUT OF
TIME.
I WANT TO GIVE THE OPPORTUNITY
TO THE THIRD PARTY COVERAGE
COMMITTEE TO ASK A QUESTION IF
THERE'S ONE BURNING.
>> HOW DO YOU TRANSLATE SOME OF
THE FINDINGS YOU GET WITH PROs
AND HOW DO YOU GET THE PATIENT
REPORTED OUTCOMES CLINICALLY?
>> IT'S A GREAT QUESTION AND
BASED ON MY EXPERIENCE DOESN'T
HAVE ONE RIGHT ANSWER.
STARTING WITH THE PAYERS OR
THOSE MAKING COVERAGE DECISIONS
UP FRONT WHAT WILL THEY NEED IN
A PARTICULAR CASE.
SOMETIMES IT'S VERY DIFFERENT
FROM THE WAY OUR RESEARCH
PIPELINE GENERALLY WORKS.
SO UNDERSTANDING WHAT TYPES OF
EVIDENCE AND FACTORS AND IT'S
NOT A ONE RIGHT ANSWER BUT A
LARGE NUMBER OF DIFFERENT KINDS
OF ENTITIES MAKING COVERAGE
DECISIONS.
SO
>> ONE MISSION IS PATIENT
REPORTED OUTCOMES.
>> NOT NECESSARILY PATIENT
REPORTED OUTCOMES.
WE'RE LOOKING AT LONG-TERM
HEALTH IMPACTS.
WE TALKED WITH PAYERS AND HAVE A
PAYER FORUM AND FREQUENTLY
TRYING TO UNDERSTAND WHERE WE
FOCUS IS ON GENERATING EVIDENCE
AND IT'S HAVING CONVERSATIONS
WITH THEM TO UNDERSTAND THE
QUESTIONS THEY'RE ASKING AND
EVIDENCE NEEDS AND IT'S NOT
ALWAYS FRANKLY NOT MOST YOU ARE
LOOKING AND COVERAGE DECISIONS
AND EMPLOYER GROUPS NOT ONLY
PURCHASERS OF HEALTH CARE AND
HAVE A BETTER ANGLE IN WHAT'S
AFFECTING EMPLOYEE POPULATIONS
AN LOSING WORK TIME AND SO HAVE
THE BROADER SPECTRUM AND HAVING
CONVERSATIONS ON THEIR NEEDS.
HOW WE APPROACH THINGS AND
DIALOGUE WITH WHAT'S HELPFUL AND
WHAT WE HEARD AS A SIMPLIFIED
STATEMENT FOLLOWED ON SHORT TERM
PRODUCT AND EVERYDAY INFORMATION
AS WELL AS LONGER TERM FINDINGS
THAT COMES THROUGH THE RESEARCH.
IT'S TAKING A COMPREHENSIVE
VIEW.
>> RATHER THAN LONG-TERM
OUTCOMES OF LYMPHATIC DISEASES
LOOK AT THE SHORT TERM
IMPLICATIONS?
>> AND THE RESEARCH TO GET THERE
AND MAY BE DIFFERENT RESEARCH IN
A STANDARD RO1 WITH A FIVE-YEAR
TIME LINE AND TRANSLATION STEP
TO GET IT TO CLINIC OR WHAT'S IN
THE CLINIC WITH RESEARCH TO BE
DONE ON A TWO-YEAR HORIZON.
>> IF YOU'RE GOING TO VALIDATE
AND TEST PATIENT REPORTED
OUTCOMES IF YOU'RE GOING TO THE
FDA AND ASK FOR DETERMINATION
EFFECTIVENESS OF A DRUG, THE END
DATE HAS A PATHWAY TO USE BUT
IT'S RIGOROUS SO KEEP THAT END
MIND.
>> IT TAKES A LONG TIME THERE'S
GROWING INTEREST IN DIGITAL
MEASURES AND DIFFERENT WAYS TO
COLLECT IN A STANDARDIZED FORMAT
THAT INFORMATION.
I THINK THE SPACE WILL CHANGE A
LOT IN THE NEXT FIVE TO 10 YEARS
BUT WHEN YOU'RE INVOLVING I HAVE
FDA INVOLVED IN OUR STEERING
COMMITTEES AND WORKING GROUPS
AND THEY ARE VERY HELPFUL AT
HELPING GET A SENSE OF REALITY.
>> I CAN DOVETAIL ON THE
QUESTION THEN, HOW DID YOU
ENGAGE THE FDA?
I'M HEARING DIFFERENT PARTS AND
EARLY ENGAGEMENT AND WHAT'S
KEEPING THEM THERE AND COMING TO
THE TABLE.
IT CREATES AN UMBRELLA AND
THERE'S POLICIES CLEARLY
DELINEATED IN THE BIOMARKER
CONSORTIUM.
WE ALLOW THEM TO BE INVOLVED IN
THE EARLY PHASE IT'S NOT SO
SPECIFIC AND NEEDS TO GO TO A
HIGHER PERSON IN THE FDA COMMENT
WHICH I THINK IS HELPFUL AND THE
EXECUTIVE COMMITTEE FDA SITS IN
THE APPROVAL PROCESS.
IT BEFORE ANY PROJECT IS
APPROVED IN A NON-VOTING
CAPACITY, FDA SITS ON IT.
>> VERY LAST QUESTION.
>> VERY SHORT AND SIMPLE ONE.
HOW MANY PEOPLE ARE INVOLVED IN
THE ORGANIZATION?
>> WE HAVE I THINK ABOUT 350
STAFF RIGHT NOW FOR ALL PARTS OF
THE ORGANIZATION.
WE'RE RUNNING OURS IS ALL
HEALTH.
WE'RE NOT FOCUSSED ON ANY
PARTICULAR DISEASE.
THAT'S OUR PROGRAMS, ITS OUR
MONITORING AND OPERATIONS TO RUN
RESEARCH ADMINISTRATION GROUP
WHICH IS ONE OF THE LARGER
GROUPS FOR THE PROCESSING AND
MANAGEMENT OF THE GRANTS.
MOST OF THOSE ARE TO THE
RESEARCH SPACE BUT THERE'S ALSO
SOME THAT HAVE TO DO WITH MAKING
AN ORGANIZATION RUN.
>> AND WE ARE 110 EMPLOYEES.
A LOT OF OUR -- WE HAVE 122
ACTIVE PROJECTS.
A LOT OF OUR PROJECT WE HAVE A
DECENTRALIZED PROJECT PROGRAM.
>> THIS IS GREAT.
THANK YOU.
OBVIOUSLY NIDDK WITH THAT I'M
GOING TO ASK ONE IMPORTANT
QUESTION, LAURA, CAN YOU PLEASE
HELP US RECRUIT WHOEVER YOU
THINK WOULD BE THE CORRECT
PERSON FOR THE PAYER FORUM
BECAUSE WE WOULD LIKE TO INVITE
THE PAYER FORUM TO HAVE THIS
CONVERSATION AS WELL AND ASK
THEIR OPINION HOW TO BASICALLY
MAKE THE LYMPHATIC DISEASES
COVERAGE BETTER.
SO DO YOU HAVE -- THIS IS NOT
THE TIME EXPECTING THE ANSWER
PLEASE THINK ABOUT WHO WOULD BE
CONTRIBUTING TO OUR DISCUSSION?
>> I WOULD ALSO ASK THE SAME FOR
THE DISSEMINATION INFORMATION
GROUP.
>> OUR COLLEAGUES HERE NOW THAT
YOU'RE MORE CLEAR WHAT WE ARE
LOOKING FOR IF YOU THINK YOU
SHOULD INVITE ANOTHER GROUP OF
PEOPLE WE ARE HAPPY BUT
TARGETING THE PAYERS.
>> I CAN FOLLOW-UP WITH STAFF
CONTACTS RELATED TO THINK ABOUT
HAVING A CONVERSATION WITH THEM
FIRST AND HOW IT MAKE CONTACTS
WITHIN THE PAYER COMMUNITY.
I DON'T KNOW IT TRANSLATES.
IT'S NOT THAT THEY'RE AN ENTITY
UNTO THEMSELVES THAT SPEAKS AND
FOCUSSED ON THE DIALING WITH
PCORI AND I'M SURE OUR STAFF
WOULD BE HAPPY TO SPEAK WITH YOU
AND THINK HOW TO MAKE THE
CONNECTIONS AND WHAT WE LEARNED
THROUGH BUILDING THOSE
CONNECTIONS.
DEFINITELY FOR DISSEMINATION AND
IMPLEMENTATION.
>> YOU CAN SEE WE HAVE A LOT OF
QUESTIONS.
NOW YOU ARE MORE FAMILIAR WITH
WHAT THE QUESTIONS ARE FOR THIS
COMMISSION AND WE WANT TO KEEP
OUR CONVERSATION GOING AND WITH
THAT I'D LIKE TO CLOSE THE OPEN
SESSION WITH OUR GRATITUDE TO
EVERYONE WHO JOINED US TODAY AS
PANELISTS AND ALSO LISTENERS ON
VIDEOCAST.
A HUGE THANK YOU TO ALL
REPRESENTATIVES OF ALL
GOVERNMENT AGENCIES AN
ORGANIZATIONS AND TO THE MEMBERS
AND CO-CHAIRS FOR THE LEADERSHIP
FOR PROMOTING DISCUSSIONS OF THE
MEET
MEETING.
SHARE INFORMATION YOU THINK IS
IMPORTANT AND THANK YOU FOR YOUR
DEDICATION AND THREW FOR THE
SUPPORT TEAM CONSISTING OF NHLBI
LEADERSHIP AND STAFF AND WORKING
GROUP AND COORDINATING COMMITTEE
AND THE WORKSHOP SUPPORT TEAM
WHO HAS BEEN INSTRUMENTAL FOR
THE MEETING.
THEY'RE THE BEST.
THEY SAY YES TO EVERYTHING.
LAST BUT NOT LEAST I WOULD LIKE
TO RELAY THE RETIREMENT.
OR CONGRATULATIONS TO YOUR
SERVICE WE MAY REACH OUT AGAIN.
WITH THAT, OPEN SESSION
ADJOURNED.
THANK YOU, EVERYONE.