WELCOME TO DAY 2 OF THE NINDS
NON-PROFIT FORUM.
I WANT TO THANK ALL SO MUCH FOR
COMING TODAY AND FOR
PARTICIPATING IN YESTERDAY'S
FABULOUS SESSION.
IT'S BEEN REALLY AMAZING TO ONCE
AGAIN GATHER IN PERSON AND
VIRTUALLY.
AND I AM DELIGHTED TO SHARE OUR
ATTENDANCE NUMBERS.
WE HAD ABOUT 340 PEOPLE TOTAL
PARTICIPATING.
WITH ALMOST 50/50, OVER 150 IN
PERSON AT DIFFERENT TIMES DURING
THE DAY YESTERDAY, AND A LITTLE
BIT OVER 180 PARTICIPATING
ONLINE.
SO I THINK THAT'S GREAT.
IT ALLOWS US TO RECONNECT IN
PERSON WHILE STILL BEING ABLE TO
INCLUDE PEOPLE FOR WHOM PERHAPS
TRAVEL IS LESS CONVENIENT OR
LESS POSSIBLE.
AND WE ALL GET TO BE PART OF THE
SAME REALLY IMPORTANT
CONVERSATIONS.
WITH THAT I'M GOING TO BRIEFLY
RECAP DAY 1, JUST AT A REALLY
HIGH LEVEL.
I WANT TO ACKNOWLEDGE THE
INCREDIBLE KEYNOTE FROM TERRY
PIROVOLAKIS.
I PRACTICED THAT.
AND HIS INCREDIBLE STORY OF THE
WAY THAT HE AND HIS FAMILY WERE
ABLE TO REALLY CATALYZE A
RESEARCH COMMUNITY AND COMMUNITY
OF DONORS AND FRIENDS AND LOVED
ONES AND EXPAND THAT AND REALLY
MOVE SCIENCE FORWARD, MOVE
TREATMENT FORWARD.
AND I THINK THAT THAT ABILITY OF
A COMMITTED PERSON, FAMILY,
CAREGIVER, TO CATALYZE RESEARCH
PROGRESS WE HEARD AGAIN AND
AGAIN YESTERDAY.
AND IT WAS INCREDIBLY INSPIRING
AND IT REMINDED ME OF THE QUOTE
ABOUT NEVER UNDERESTIMATING THE
POWER OF A SMALL GROUP OF
COMMITTED PEOPLE TO CHANGE THE
WORLD.
INDEED, IT IS THE ONLY THING
THAT EVER HAS.
I JUST REALLY LEFT YESTERDAY'S
SESSION WITH THE WORDS OF PENNY
DAKS IN MY HEAD, THE HOPE, WE
HAVE THESE TREATMENTS FOR RARE
AND ULTRA RARE CONDITIONS, AND
THAT REALLY IS A RESULT OF THE
RESEARCH COMMUNITY, THE
PARTNERSHIP DEVELOPED, AMONG
RESEARCHERS THEMSELVES,
WILLINGNESS TO COLLABORATE WITH
EACH OTHER, WITH INDUSTRY, WITH
GOVERNMENT, WITH NIH AND FDA AND
OTHERS.
FOR THE NON-PROFITS TO PARTNER,
WE HAD A SESSION ABOUT HOW
NON-PROFITS FUND RESEARCH AND
ONE OF THE KEY HIGHLIGHTS THERE
WAS THE IMPORTANCE OF
PARTNERSHIPS AND COLLABORATIONS
THERE TOO.
I WANT TO ACKNOWLEDGE SOME OF
THE CHALLENGES THAT WERE RAISED
IN YESTERDAY'S SESSION.
IT IS CLEAR THAT WHILE A SMALL
GROUP OF COMMITTED PEOPLE CAN DO
TRULY REMARKABLE THINGS, IT'S
ALSO CLEAR IT COMES AT A GREAT
COST.
IT COMES AT GREAT PERSONAL COST,
GREAT FINANCIAL COST, COMES AT A
GREAT COST IN TERMS OF TIME AND
SOMETIMES ALTHOUGH THE FIELD IS
MOVED FORWARD, THEIR OWN FAMILY
DOES NOT DIRECTLY BENEFIT FROM
THAT, AND THAT IS ALSO A COST
THAT WE HAVE TO ACKNOWLEDGE.
AND I ALSO WANTED TO REALLY
ACKNOWLEDGE THE ISSUES BROUGHT
UP ABOUT THINKING ABOUT EQUITY
TO ACCESS, AS THIS HOPE IS
SWELLING AND TREATMENTS ARE
ACTUALLY BEGINNING TO EMERGE, I
THINK THAT IT IS WORTH THINKING
ABOUT HOW TO MAKE SURE THAT
THESE TREATMENTS CAN BE
AVAILABLE MORE WIDELY.
THAT'S A LITTLE BIT OUT OF SCOPE
OF NIH AND THOSE GATHERED HERE
TODAY, BUT I THINK IT'S
SOMETHING THAT WE ALL NEED TO
STAY COMMITTED TO.
AS WE LOOK AHEAD TO TODAY'S
DISCUSSIONS WE'RE GOING TO HAVE
A FABULOUS PANEL DISCUSSION FOR
PATIENT DATA AND FOCUSED ON
COMMUNITY ENGAGEMENT STRATEGIES
TO BOOST DIVERSITY ENROLLMENT IN
CLINICAL TRIALS FROM A PHARMA
INDUSTRY PERSPECTIVE.
I'M VERY EXCITE AND LOOKING
FORWARD TO THAT.
QUICKLY, BEFORE I INTRODUCE THE
FIRST PANEL, I DID WANT TO SHARE
WITH THIS GROUP IN PARTICULAR
STEPS THAT NIH, NINDS IN
PARTICULAR, HAVE BEEN TAKING TO
REALLY LEAN IN TO PARTNERING
WITH PEOPLE WITH LIVED
EXPERIENCE OF DECEMBER.
DISEASE.
WE HIGHLIGHTED IMPORTANCE OF
INCREASING PATIENT ENGAGEMENT IN
NINDS RESEARCH TO BETTER ADDRESS
PRIORITIES OF PATIENTS AND
FAMILIES AND IMPROVE EFFICIENCY
AND EFFECTIVENESS OF RESEARCH.
I WANT TO EMPHASIZE NOT ONLY
DOES THIS FEEL LIKE THE RIGHT
THING TO DO; THE MAKES THE
RESEARCH BETTER, MAKES IT MOVE
FASTER.
I THINK WE SAW THAT AGAIN AND
AGAIN YESTERDAY, IN A WAY THAT
REALLY BRINGS A LOT OF HOPE
LOOKING FORWARD AS NINDS IS
LOOKING FOR WAYS TO DO THIS MORE
AND MORE EFFECTIVELY.
A FEW EXAMPLES OVER THE LAST ONE
TO TWO YEARS, STEPS WE HAVE
TAKEN TO REALLY LEAN IN TO THIS
ENGAGEMENT, WE'RE ENGAGING WITH
PEOPLE WITH LIVED EXPERIENCE
MORE INTENTIONALLY AROUND
SETTING RESEARCH PRIORITIES FOR
THE SCIENTIFIC COMMUNITIES,
SETTING SCIENTIFIC PRIORITIES
FOR ALS, WE WORKED CLOSELY WITH
PEOPLE WITH LIVED EXPERIENCE, ON
OUR STEERING COMMITTEE, ON ALL
FIVE OF THE WORKING GROUPS.
THERE WERE NUMEROUS
OPPORTUNITIES FOR THE
INCORPORATION OF PUBLIC INPUT
AND INCLUSION OF THESE
INDIVIDUALS IN OUR DISCUSSIONS
DEEPENED THEM AND YIELDED A
RICHER SET OF SCIENTIFIC
PRIORITIES.
I WANT TO NOTE THAT THERE WAS AN
INTENTIONAL EFFORT FOR THE PLAN
THAT EMERGED TO INCLUDE PLAIN
LANGUAGE EXPLANATIONS OF
SCIENTIFIC PRIORITIES WHICH I
THINK IS REALLY AN IMPORTANT
PIECE OF SHARING WITH ALL OF OUR
STAKEHOLDERS THE IMPORTANCE OF
RESEARCH PRIORITIES AND HOW THEY
ARE SET.
LOOKING AHEAD, OUR ME/CFS ROAD
MAP INCLUDES PEOPLE WITH LIVED
EXPERIENCE AT THE PLANNING
PROCESS, I'M EXCITED TO SEE WHAT
THE PROCESS YIELDS.
WE'RE ENGAGING WITH PEOPLE WITH
LIVED EXPERIENCE TO INFORM
RESEARCH IN THE FIELD, HAD A
SERIES OF ROUNDTABLES AROUND
PARKINSON'S DISEASE AND
DEVELOPING THERAPEUTICS AND A
BRAIN INITIATIVE GROUP LOOKING
AT TRIAL RESPONSIBILITIES,
SHARING INDIVIDUAL LEVEL HUMAN
BRAIN DATA, AND THE DISCUSSIONS
INCLUDED PEOPLE WITH LIVED
EXPERIENCE SO THEIR PERSPECTIVES
WERE PART OF THE CONVERSATION AT
EVERY POINT.
THEY DIDN'T JUST PROVIDE A
REACTION AT THE END.
WE'RE ALSO LOOKING FOR WAYS TO
ENCOURAGE ENGAGEMENT WITH LIVED
EXPERIENCE ACROSS NIH AND
BEYOND.
WE'VE BEEN SHARING STRATEGIES
AND LEARNING FROM OTHERS AT A
RECENT INTERAGENCY COLLABORATIVE
TO ACCELERATE RESEARCH ON
EPILEPSY MEETING, WE HAD A
SESSION ON STRATEGIES AROUND
ENGAGING WITH PEOPLE WITH LIVED
EXPERIENCE AT THE RECENT BRAIN
INITIATIVE CONFERENCE, AND WE
HELD A WORKSHOP AT THE NATIONAL
SCIENCE ASSOCIATION.
I JUST WANT TO ACKNOWLEDGE THE
ONGOING WORK THAT HAS BEEN GOING
ON IN THE HELPING TO END
ADDICTION LONG-TERM INITIATIVE,
NIH HEAL INITIATIVE, WHICH IS
FOCUSED ON ENDING THE OPIOID USE
DISORDER EPIDEMIC AND DEVELOPING
MORE EFFECTIVE AND NON-ADDICTIVE
PAIN STRATEGIES AND THERAPIES.
THERE ARE EFFORTS TO ENGAGE
PEOPLE WITH LIVED EXPERIENCE IN
THE ADVISORY STRUCTURE THROUGH
"HEAL" COMMUNITY PARTNERS
COMMITTEE AND OPPORTUNITIES, FOR
EXAMPLE, FOR FUNDING SUPPLEMENTS
THROUGH "HEAL" TO BRING IN
PEOPLE WITH LIVED EXPERIENCE AND
ALSO TO DIVERSIFY PARTICIPATION
IN A NUMBER OF DIFFERENT
"HEAL"-RELATED ACTIVITIES, JUST
A FEW EXAMPLES OF THE WORK
"HEAL" HAS DONE IN THIS SPACE.
SO, A LOT OF OUR EFFORTS ARE THE
RESULTING THE REALLY PASSIONATE
ENGAGEMENT OF STAFF AT ALL
LEVELS ACROSS NINDS.
PEOPLE WORK REALLY, REALLY HARD
AT THIS INSTITUTE TO TRY TO MAKE
A DIFFERENCE FOR THE PATIENTS.
AND I'M JUST REALLY EXCITED TO
SHARE THAT WE WERE ABLE TO
RECRUIT A LITTLE OVER A YEAR AGO
DR. REBEKAH CORLEW, SHE'S
PARTNERING TO MOVE THESE
TRAJECTORIES FORWARD.
IF YOU HAVE SUGGESTIONS OR YOU
WANT TO ENGAGE WITH HER TO FIND
INFORMATION OUT, ACROSS THE
INSTITUTE, PLEASE DO SO.
AND FINALLY IT IS MY PRIVILEGE
TO INTRODUCE OUR NEXT SESSION.
SO, AGAIN, THIS IS A PANEL
DISCUSSION ON PATIENT DATA.
I WOULD LIKE TO INTRODUCE OUR
MODERATOR, CHRISTINE VON
RAESFELD.
SHE'S FOUNDER AND CEO OF PEOPLE
WITH EMPATHY, A PILLAR OF
PATIENT ADVOCACY AND ALLYSHIP IN
THE RARE AND CHRONIC DISEASE
COMMUNITY.
THROUGH THE LENS OF HER LIVED
EXPERIENCE SHE HAS BECOME A
CHAMPION FOR PATIENT VOICE,
DIVERSITY, INCLUSION IN CLINICAL
RESEARCH, EQUITABLE PATIENT
PARTNERSHIP, BROUGHT A UNIQUE
PERSPECTIVE RANGING FROM
CLINICAL RECRUITMENT TO DATA,
AND DIGITAL RIGHTS FROM A TRUE
PATIENT PERSPECTIVE.
SHE SERVED AS e-PATIENT
SCHOLAR WITH THE "ALL OF US"
PROGRAM AND EXPERT PANELIST WITH
CENTERS FOR MEDICARE AND
MEDICAID SERVICE, CMS, AMONG
OTHER ROLES.
WE'RE PRIVILEGED TO HAVE HER
HERE TODAY.
>>I'M GOING TO
INTRODUCE -- WELL, NOT REALLY
INTRODUCE MY PANELISTS BECAUSE I
FIGURE YOU CAN LOOK US ALL UP ON
LINKED IN, WE DON'T NEED TO GO
INTO A HUGE -- SORRY.
WE DON'T NEED TO GO INTO A HUGE
DISCUSSION ON WHAT EVERYONE DOES
BUT I WILL HAVE MY PANELISTS
EACH INTRODUCE THEMSELVES WITH A
QUESTION.
AND SO I'M GOING TO -- DIANE,
YOU'RE THERE ONLINE.
WE'LL GO TO YOU FIRST.
MY QUESTION THAT I WANT YOU GUYS
TO ANSWER FOR OUR AUDIENCE IS
HOW DO YOU BELIEVE RESPONSIBLE
DATA SHAKER CAN POSITIVELY
IMPACT HEALTH CARE RESEARCH,
PATIENT CARE AND INNOVATION
WHILE ENSURING PRIVACY AND
OWNERSHIP RIGHTS ARE RESPECTED.
SO, DIANE, DO YOU WANT TO GO
FIRST?
>>GREAT.
THANK YOU.
I'M SO HAPPY TO JOIN THIS
ESTEEMED PANEL, SO SORRY I CAN'T
BE THERE IN PERSON.
I'M TRAINED AS A NEUROSCIENTIST
FOR 30 YEARS, CRITICAL PATH
INSTITUTE, THE QUESTION YOU POSE
IS WHY I CAME TO THIS AMAZING
ORGANIZATION.
MY CAREER WAS IN LARGE
PHARMACEUTICAL COMPANIES TRYING
TO GET THERAPIES TO PATIENTS AND
LOSING TWO FAMILIES WITH
DISEASES I WORKED ON.
THAT CRITICAL PATH INSTITUTE,
COLLABORATIONS ACCELERATE DRUG
DEVELOPMENT, ADVANCING BETTER
TREATMENT WORLDWIDE.
AND WHAT WE'VE DONE, WHAT IS SO
REWARDING, IS TO -- THANKS TO
GENEROUS FUNDING FROM FDA BRING
DATA TOGETHER, PATIENT LEVEL
DATA, FROM OBSERVATIONAL STUDIES
AND CLINICAL TRIALS TO INFORM
NEW TOOLS, THAT'S REALLY DRIVING
CHANGE, HELPING TO EMPOWER THIS
OF THE SUCCESS AND NEW APPROVAL
FOR DRUG TREATMENTS NOW IN A
VARIETY OF DISORDERS.
>>HI, ALISA FINK, NATIONAL
ORGANIZATION FOR RARE DISORDERS.
I'M EXCITED TO BE HERE TODAY.
TO ANSWER YOUR QUESTION, THE WAY
THAT I THINK ABOUT IT IS THIS
WORK THAT WE'RE ALL INVOLVED IN
COLLECTIVELY IS BIG AND IT'S
HARD.
AND REALLY, WE NEED ALL OF US TO
GET TO BE THINKING ABOUT IT
COLLECTIVELY AND TOGETHER.
DATA COLLECTION TAKES TIME,
TAKES MONEY, TAKES RESOURCES,
AND IT'S PRECIOUS.
AND THE OPPORTUNITY THAT IT
PROVIDES WILL BE STRENGTHENED
AND BUILT UPON IF WE'RE ALL
COMING AT THIS DATA WITH OUR
UNIQUE EXPERIENCES, EXPERTISE,
AND PERSPECTIVES, AND THOSE ARE
THE THINGS THAT ARE GOING TO
MAKE THIS DATA VALUABLE.
WE'RE ALL GOING TO BRING
SOMETHING DIFFERENT TO IT, AND
HELP TO REALLY MAKE THAT DATA
IMPACTFUL AND MEANINGFUL,
WHETHER IT'S FOR RESEARCH, THE
DRUG DEVELOPMENT PATHWAY, AND
IMPROVING HEALTH CARE.
>>WOW, IT'S GOING TO BE TOUGH
TO ADD TO ANY OF THOSE COMMENTS.
BUT MY NAME IS DAVE VOCCOLA,
INTEGRATED HEALTH PRACTICES
FOCUSES ON TECHNOLOGY AND DATA
INITIATIVES FOR NON-PROFITS.
I THINK THE ONLY WAY TO EVEN ADD
TO WHAT MY COLLEAGUES HAVE
ALREADY SAID IS THAT I SEE THE
VALUE OF PATIENT DATA
ESSENTIALLY EQUALING, IN SOME
CASES ENHANCING, THE SCIENCE IN
THE NEXT ERA OF DRUG DISCOVERY
AND TREATMENT DISCOVERY, AND
PRECISION MEDICINE.
I'VE BEEN THINKING ABOUT THIS
DATA, EVEN BEFORE I CO-FOUNDED A
COMPANY THAT JUST BUILT PATIENT
REGISTRIES FOR NON-PROFITS AND
TRIED TO LEVEL THE PLAYING FIELD
A BIT IN TERMS OF GETTING SOME
DATA OUT OF THE RESEARCH SITES
AND REALLY ENHANCE THAT PATIENT
VOICE AND GET IT -- THAT'S WHAT
ALL OF US HAVE IN COMMON ON THIS
PANEL.
BUT I THINK THE NEW TECHNOLOGIES
AND NEW ABILITIES FOR PATIENTS
TO FULLY ENGAGE AND FOR THEIR
FAMILIES AND CAREGIVERS TO FULLY
ENGAGE IN THE RESEARCH
ENTERPRISE, THAT'S GOING TO BE
THE DEFINING DIFFERENTIATOR
BETWEEN THE LAST AND CURRENT
GENERATION OF RESEARCH.
IT BEHOOVES ALL OF US TO
IDENTIFY THE RIGHT PATHWAYS,
WHETHER IT BE FUNDING OR
PARTNERSHIP PATHWAY, TO ENSURE
THAT THE MAIN SERVICE THAT
NON-PROFITS ARE BRINGING ARE
BRINGING TO THEIR COMMUNITIES, I
HOPE THIS ISN'T TOO
CONTROVERSIAL BUT I THINK
NON-PROFITS AND PATIENT
ORGANIZATIONS ARE BY FAR THE
BEST STEWARDS FOR THAT
PATIENT-LEVEL DATA THAT'S GOING
TO BE GENERATE AND NEEDS TO BE
CONSOLIDATED.
CERTAINLY BETTER THAN MANY
FOR-PROFITS AND NICHE
INVESTMENTS.
I DO GET EXCITED ABOUT THESE
TYPES OF DISCUSSIONS.
>>I'LL TELL YOU REALLY QUICK WHY I
THINK DATA IS IMPORTANT AND
WE'LL GO TO OUR QUESTIONS.
WE'LL HAVE THIS AS A
CONVERSATIONAL STYLE.
WE DON'T WANT TO FOCUS ON ONE
ORGANIZATION.
WE'VE GOT THREE GREAT
ORGANIZATIONS HERE WHO KNOW WHAT
THEY ARE DOING AND WHO CAN OFFER
ADVICE TO THOSE OF YOU OUT
THERE.
FOR ME, I'M A PATIENT, ON
MEDICARE RIGHT NOW.
I'VE BEEN -- I'M SORRY, 20 YEARS
ON MEDICARE, AND I'M UNDIAGNOSED
STILL.
MULTIPLE RARE DISEASE AND
AUTOIMMUNE.
MY DATA HAS BECOME EXTREMELY
IMPORTANT IN TRYING TO FIND MY
UNDERLYING CONDITION.
IN THE NEW DIGITAL AGE THAT
WE'RE COMING UPON, I SEE A GREAT
NEED FOR PATIENTS TO UNDERSTAND
DATA, HOW IT'S COLLECTED, AND
WHY IT'S VALUABLE TO REALLY TAKE
ADVANTAGE OF THAT AND BENEFIT
OUR COMMUNITIES.
SO I'M GOING TO START WITH SOME
QUESTIONS.
OUR FIRST QUESTION HERE IS, WE
REALLY JUST WANT TO KIND OF
DEFINE PATIENT DATA FOR YOU
BECAUSE IT IS A VAST AMOUNT OF
THINGS FROM WEARABLE DEVICES,
WHAT'S THE DIFFERENCE BETWEEN
PATIENT DATA AND EVEN AGGREGATE
DATA.
SO, DIANE, YOU'RE ON OUR SCREEN
SO I'LL GO TO YOU FIRST.
I DON'T WANT TO DO THIS
WEIRD-LOOKING BACK AND FORTH.
>>YES, THANK YOU SO MUCH FOR
THAT QUESTION.
IT'S SO TIMELY.
WHAT WE'VE DONE AT CRITICAL PATH
INSTITUTE WITH GENEROUS FUNDING
FROM THE FDA, BRING TOGETHER
DATA FROM OBSERVATIONAL STUDIES
AND CLINICAL TRIALS, MANY HAVE
FAILED IN THE PAST.
SO, FOR EXAMPLE, THE CONSORTIA
THAT I CURRENTLY LEAD NOW,
PARKINSON'S CONSORTIA BROUGHT
TOGETHER CLINICAL TRIAL DATA
FROM 18 TRIALS AND THE TOTAL
NUMBER OF STUDIES THAT WE HAVE
IS -- TOTALS OVER 13,000
INDIVIDUALS.
THIS DISEASE WITH SUCH AN
EXCITING PLETHORA OF NOVEL
TARGETS COMING FORWARD WE NEED
TO UNDERSTAND THIS DISEASE IN
NEW WAYS, THANKS TO THE TYPE OF
PATIENT DATA THAT'S NEEDED FOR
REGULATORY SUCCESS.
AND WHEN YOU THINK ABOUT
AGGREGATE DATA, IT'S INFORMATIVE
TO LEARN, WE WANT TO LOOK AT
PATIENT-LEVEL DISEASE.
WE'RE FORTUNATE TO HAVE STRONG
PARTNERSHIPS WITH NINDS, THE
DATA THEY BRING FORWARD IS ALSO
CRITICAL, IT BE DONE AT THE
PATIENT LEVEL SO WE CAN REALLY
DECIPHER WHAT GENES AND WHAT
ENVIRONMENTAL FACTORS CAN BE
USED TO DEFINE PATIENTS IN
FUTURE TRIALS WITH PRECISION
MEDICINE STRATEGIES.
SO WE REALLY NEED TO LOOK AT
THAT INDIVIDUAL PATIENT-LEVEL
DATA FOR SUCCESS.
>>DO EITHER OF THE TWO OF YOU
WANT TO TAKE IT?
DAVID, I THINK YOU'RE MORE
AGGREGATE.
>>I THINK THERE'S TWO WAYS OF
THINKING ABOUT PATIENT DATA.
THERE'S SORT OF DATA ABOUT
PATIENTS, AND DATA FROM
PATIENTS.
AND I TEND TO SPEND MORE TIME
SORT OF IN THE SECOND SPACE,
BECAUSE NORD RUNS THE "I AM RARE
PROGRAM," REALLY ABOUT PATIENT
ADVOCACY GROUPS, SPONSORING,
DESIGNING, IMPLEMENTING NATURAL
HISTORY STUDIES THAT ARE BASED
ON DATA FROM PATIENTS.
SO THAT'S REALLY THE SPACE WHERE
WE'RE REALLY THINKING ABOUT IT,
WHAT IS IT THAT PATIENTS KNOW
ABOUT THEIR LIVED EXPERIENCE
THAT CAN CONTRIBUTE AND REALLY
BE MEANINGFUL AS WE THINK ABOUT
OUR UNDERSTANDING OF THE
DISEASE.
>>WE'RE STILL SHARING.
SO I THINK FROM PATIENT DATA
PERSPECTIVE, ABSOLUTELY AGREE
WITH EVERYTHING THAT'S BEEN SAID
ALREADY.
I THINK THE PLACE FOR ME THAT'S
MOST EXCITING IS FOR MOST OF OUR
CAREERS WHEN WE THOUGHT ABOUT
PATIENT DATA, IT ALWAYS HAD THIS
CONNOTATION OF LIVED EXPERIENCE
OR PATIENT-REPORTED OUTCOME DATA
AND WENT IN THIS BOX OF THINGS
WE GET FROM THE PATIENTS.
AND THEN A LOT OF THE HARD DATA
CAME FROM THE RESEARCH SITES AND
CAME FROM OTHER PLACES.
I THINK THAT TRANSITION IS
REALLY EMBLEMATIC OF THE OVERALL
TRANSITION TO WHERE EVEN WHEN WE
THINK ABOUT WHAT PATIENTS CAN
SHARE, IT'S NO LONGER JUST THEIR
LIVED EXPERIENCE OR JUST
RESPONDING TO A PRO.
THANKS TO ONE OF THE PLACES THE
GOVERNMENT DOESN'T GET ENOUGH
CREDIT, THE LONG TAIL OF
INVESTMENTS MADE FROM THE
RECOVERY ACT THROUGH THROUGH THE
2016 CURES ACT, AND THE RELATED
ADVANCEMENTS IN THE INTERIM, WE
NOW ARE ABLE TO GIVE PATIENTS
ACCESS TO THEIR OWN DATA.
THEY ARE ABLE TO RETRIEVE THAT
ELECTRONICALLY, SHARE WITH
PEOPLE THEY TRUST.
SO I THINK THAT'S VERY MUCH
CHANGING THE LANDSCAPE OF WHAT
WE AS A COMMUNITY THINK MUCH AS
PATIENT DATA, AND THINK OF AT
DATA THAT IS ACCESSIBLE TO
NON-PROFITS.
WE NO LONGER ARE BEHOLDEN TO
SITE NETWORKS IF WE WANT TO GET
CLINICAL DATA, NO LONGER
BEHOLDEN TO CLINICAL
PARTNERSHIPS TO LEARN WHAT'S
HAPPENING IN THE DOCTOR'S OFFICE
AS OPPOSED JUST WHAT IS
HAPPENING AT HOME.
THAT INVERSION IS ALSO EXPOSING
TO LIFE SCIENCE COMPANIES AND
OTHER PARTNERS THAT, WAIT A
SECOND, WE DON'T HAVE TO TREAT
THE PATIENT AS A BY-PRODUCT OF
RESEARCH.
WE CAN TREAT THEM AS THE CENTER
OF IT.
OUR DATA BECOMES A WAY TO
AUGMENT WHAT WE'RE ABLE TO
ACHIEVE DIRECTLY FROM PATIENTS.
NOW WE HAVE A NATURAL PATHWAY TO
BRING TOGETHER THINGS LIKE WHAT
DIANE WAS TALKING ABOUT,
TYPICALLY DE-IDENTIFIED DATA AT
THE PATIENT LEVEL AND MARRY THAT
WITH DIRECT TO PATIENT
INITIATIVES THAT ARE PLACING
THOSE IDENTIFIED FOLKS RIGHT AT
THE CENTER, AND NOW ANYTHING
THAT MAY BE RELATED TO THEIR
HEALTH, ENVIRONMENTAL FACTORS,
DATA THEY CAN SHARE FROM A
WEARABLE, DATA THEY CAN RESPOND
TO, THAT THEY HAVE OBSERVED IN
THE FAMILIES CAN COME TOGETHER
AS A 360-DEGREE VIEW.
THE DEFINITION IS HARD BECAUSE
IT'S EXPANDED DRAMATICALLY OVER
THE YEARS.
>>YOU MENTIONED TRUST AND THE
HOLISTIC CARE BECAUSE I THINK
WE'VE GOTTEN SO SILOED IN OUR
VIEW OF EVERYTHING THAT WE
REALLY DON'T PUT THINGS TOGETHER
TO FIGURE THESE THINGS OUT IN A
WAY WE SHOULD.
WE HAVE A COUPLE DIFFERENT
ORGANIZATIONS HERE.
ONE OF THE THINGS I WANTED TO
ASK YOU IS WE HAVE MULTIPLE
STAKEHOLDERS UP HERE.
WHAT ARE THE PRIMARY INTERESTS
AND PRIORITIES AND CONCERNS FOR
EACH OF OUR STAKEHOLDERS UP
HERE?
FOR ME, I WILL TELL YOU THE DATA
IS MY WAY OF SOLVING THINGS.
I FEEL LIKE THE ONLY PERSON WITH
ENOUGH INCENTIVE TO FIX ME IS
ME.
SO HOW WE AGGREGATE THAT DATA IS
ONE OF MY POINTS OF INTEREST.
I WOULD LOVE TO HEAR FROM THE
THREE OF YOU.
DIANE, DO YOU WANT TO GO FIRST?
>>YES.
THANK YOU SO MUCH.
ONE OF THE MOST REWARDING THINGS
ABOUT MY ROLE AT CRITICAL PATH
INSTITUTE IS BRINGING DIVERSE
STAKEHOLDERS TOGETHER.
WHEN WE STARTED, WHEN I CAME,
THE IDEA OF DIFFERENT GROUPS
WORKING TOGETHER WAS QUITE
FOREIGN, ESPECIALLY DIFFERENT
COMPANIES THAT WERE COMPETING TO
TRY TO GET THERAPIES APPROVED.
NOW IT'S A WHOLE NEW WORLD,
RIGHT?
I WANT TO THANK THE FDA
ESPECIALLY FOR THEIR LEADERSHIP
AND PATIENT-FOCUSED DRUG
DEVELOP.
BUT BRINGING TOGETHER
STAKEHOLDERS, PUTTING THE
PATIENT FRONT AND CENTER TO THE
NETWORK AND ECOSYSTEM IS RATHER
NEW.
IN INDUSTRY WE DIDN'T EVEN HAVE
THAT CONVERSATION WHEN I WAS IN
THE LAB ABOUT PATIENT-CENTERED
DRUG DEVELOPMENT AND HOW THESE
NOVEL TARGETS NEEDED TO REALLY
LOOK AT WHAT'S CLINICALLY
MEANINGFUL TO PEOPLE IN THEIR
LIVES.
FOR ME, THE BIGGEST EARLIEST
STAKEHOLDER GROUP THAT WAS
REALLY INHERENTLY DIFFICULT TO
GRASP WHAT SUCCESS LOOKS LIKE IN
THIS DATA SHARING SPACE IS THE
ACADEMICS, AND THIS IS WHERE THE
NINDS COMES IN TO HELP BRIDGE
THAT.
AND OUR PARTNERS IN EUROPE AS
WELL, BECAUSE, AGAIN, THAT
INCORPORATION TUITIONAL
GOVERNANCE AND HISTORY IS
ACADEMIC ADVANCE THROUGH
INDIVIDUAL SUCCESSES BY
PUBLISHING PAPERS AND GETTING
TENURE TRACK, THE WORLD HAS
CHANGED.
OBVIOUSLY INDUSTRY BEING AT THE
TABLE, WITH PAYERS NOW AND THE
REGULATORS ARE EARLY AND OFTEN
FULLY ENGAGED IN WHAT WE DO.
EVERYTHING WE DO AT C-PATH IS
UNDER ADVISEMENT OF WORLDWIDE
REGULATORY AGENCIES.
HAVING THE PATIENTS DRIVE
EVERYTHING IS NEW AND EVOLVING
BUT I'M SO REWARDED.
THIS MEETING IS SUCH A TESTIMONY
TO WHAT'S HAPPENED AND HOW
TRANSFORMATION IS TAKING PLACE.
>>WE ALL LOVE TO HEAR THAT.
DAVID, DO YOU WANT TO GO NEXT?
>>SURE.
I HAVE A SLIGHTLY MAYBE UNIQUE
PERSPECTIVE SITTING IN A VERY
LARGE CRO, BUT SITTING WITHIN A
PART OF THAT CRO THAT'S
DEDICATED TO SERVING
NON-PROFITS.
SO I'D LIKE TO THINK THAT
PURVIEW OF BEING ABLE TO SEE
ESSENTIALLY WHAT'S HAPPENING IN
LIFE SCIENCES AS WELL AS WHAT
WE'RE SEEING IN HEALTH CARE
ORGANIZATIONS AND NON-PROFIT
SPACE, WE TRY TO BRING
STAKEHOLDERS TOGETHER NOT SO
MUCH DIRECTLY.
WE DO THAT SOMETIMES.
BUT WE SEE OUR ROLE AS PRIMARILY
FOCUSING ON WHERE THE HIDDEN
BEST PRACTICES THAT WOULD NEVER
ACTUALLY BE SHARED ACROSS THE
DIFFERENT STAKEHOLDERS GROUPS
BECAUSE OF THEIR NATURAL ROLES
IN THE RESEARCH ENTERPRISE.
WHETHER THAT'S TECHNOLOGY BASED
OR PATIENT-FACING, YOU KNOW, WE
SEE OURSELVES TRYING TO SAY,
HEY, YOU MAY NOT BE AWARE THAT
NON-PROFITS CAN DO THIS NOW
BECAUSE YOUR EXPERIENCE
PREVIOUSLY HAS BEEN X, AND IT
WAS LIMITED TO THIS BOX.
BUT I'M ON PROJECTS THAT HAS
THEM PUTTING TOGETHER PLATFORMS
CAPABLE OF GOING BEYOND IN THE
HISTORY OR JUST BEING PART OF
IT, I THINK THAT'S BEEN A
BENEFICIAL PART OF MY JOB,
SEEING ACROSS THE BOARD AND THEN
SAY LET ME JUST CLARIFY THAT
ASSUMPTION FOR YOU BECAUSE
THERE'S BETTER PARTNERSHIPS THAT
CAN BE HAPPENING HERE.
>>I LOVE THAT YOU'RE SAYING
THAT.
I LOVE THAT THE CROs HAVE
TAKEN A DIFFERENT STANCE.
EIGHT YEARS AGO WHEN I STARTED
INTERACTING WITH CROs I ASKED
HOW WE INVOLVE PATIENTS.
WE DON'T.
THAT'S UP TO OUR SPONSORS.
THAT HAS CHANGED, AND I'M
EXCITED FOR IT.
>>SURE, NOT MUCH TO ADD.
WE REALLY TALKED ABOUT THIS BUT
I THINK THAT'S THE CHANGE THAT
WE'RE SEEING THAT WE'RE THINKING
ABOUT STAKEHOLDERS, IN THIS
REALLY BROAD WAY.
REGULATORS, PAYERS, PATIENTS,
FAMILY MEMBERS, THINKING ABOUT
BRINGING EVERYBODY TOGETHER IN
WAYS WE HAVEN'T.
TO REALLY START DOING SOME OF
THAT WORK TO UNDERSTAND WHERE
THERE ARE TENSION POINTS AND
WHAT DO GROUPS NEED SO WHAT
WE'RE BUILDING AND DESIGNING CAN
MEET THE NEEDS OF ALL OF THESE
GROUPS MOVING FORWARD.
>>WE COVERED MOST.
I WANT TO MOVE ON BECAUSE THIS
IS A VAST LANDSCAPE FOR DATA.
LET'S TALK ABOUT THE PROGRESS
THAT HAS BEEN MADE IN DATA.
DAVID, I KNOW YOU WANTED TO
MENTION A FEW KEY POINTS.
WE'LL START WITH YOU AND THEN,
DIANE, WE'LL GO TO YOU NEXT.
>>YEAH, I ALLUDED TO SOME.
I THINK THAT IF YOUR JOB ISN'T
TO NERD OUT ABOUT THE PROGRESS,
IT'S REALLY EASY TO MISS IT.
BUT IT'S -- IN THE GRAND SCHEME
OF THINGS THERE'S BEEN A METHOD
TO THE MADNESS WHERE INVESTMENTS
ARE MADE, TAKING THE U.S. QUITE
FRANKLY FROM A PUNCHLINE WHEN IT
CAME TO DATA INTRAOPERABILITY TO
THE FRONT OF THE PACK.
WHEN I WORKED WITH MY
INTERNATIONAL COLLABORATORS I
SAY LET'S DO IT THIS WAY,
CONVERSATIONS BEFORE THAT WERE
THE OTHER WAY AROUND.
THE CRUX WAS THE 21ST CENTURY
CURES ACT, THE DUAL INITIATIVES
BY CMS AND OMC TO NOT ONLY
MANDATE INTRATRAY BUILT TO OF
BUT OF -- INTRAOPERABILITY, BUT
EMPOWER THE PATIENTS AND MAKE IT
CLEAR PATIENTS DO OWN THEIR DATA
AND THEY ARE THE STAKEHOLDERS
THAT MATTER MOST IN THIS
CONVERSATION.
AND THAT IF THEY DECIDE DATA
SHOULD GO SOMEWHERE ELSE, IT
GOES SOMEWHERE ELSE.
AND THAT WAS JUST NOT THE CASE
FOR OUR ENTIRE LIVES.
SO I THINK THAT PROGRESS ALONE
PAIRED WITH THE TECHNOLOGY
DECISIONS THAT ONC MADE, WHERE
THEY TOOK A STAND AND SAID WE'RE
GOING TO GO WITH HL 7 FHIR,
GOING TO MANDATE THINGS SPECIFIC
INSTEAD OF BROAD AND EVERYBODY
CAN IMPLEMENT WHAT THEY WANT,
WE'RE ALREADY SEEING THE
BENEFITS OF THAT AND EVEN IF WE
HAVE SMARTPHONES CAN NOW
DOWNLOAD OUR RECORDS.
THAT'S THANK TO A LOT OF THAT
WORK AND THE FACT WE CAN NOW
SHARE WITH NON-PROFITS, THANKS
TO THAT GOVERNMENT WORK.
>>I WOULD SAY ON DATA OWNERSHIP
WE'RE NOT QUITE THERE YET.
OWNERSHIP I THINK IS A TERM THAT
AS PATIENTS, I WORK WITH A GROUP
LIKE COLLECTIVE, WE LIKE DIGITAL
RIGHTS, BECAUSE OWNERSHIP
IMPLIES YOU CAN SELL SOMETHING.
WE DON'T WANT TO PUT THAT IN
THERE.
DIANE, WHAT ARE YOUR THOUGHTS?
>>YEAH, PROGRESS TO ME IS JUST
GAME CHANGING.
AGAIN, I HAVE A LONG HISTORY OF
DRUG DEVELOPMENT.
WHEN I CAME TO CRITICAL PATH
INSTITUTE 12 YEARS AGO WE HAD
FIVE PROGRAMS.
NOW WE HAVE OVER 28 DIFFERENT
PROGRAMS ACROSS MULTIPLE
DISEASES.
THIS IS NINDS.
SO MANY OF THESE DISORDERS SPAN
NERVOUS SYSTEM DISORDERS BUT AN
EXAMPLE OF WHAT'S TRANSFORMED
JUST IN THE PAST FIVE YEARS IS
IN THE AREA OF ALZHEIMER'S
DISEASE.
JUST FIVE YEARS AGO OUR
INTEGRATED DATABASE LED BY THE
CRITICAL PATH FOR ALZHEIMER'S
DISEASE CONSORTIUM HAD ABOUT
35,000 INDIVIDUAL PATIENT-LEVEL
DATA RECORDS FROM 35,000
PATIENTS.
AS OF TODAY WE'VE PRESENTED LAST
WEEK BY OUR PARTNERS AND LEADERS
AT C-PATH, WE HAVE OVER 100,000
INDIVIDUAL PATIENT RECORDS IN
OUR INTEGRATED DATABASE, MANY
FROM CLINICAL TRIALS INCLUDING
BIOMARKER DATA WHICH IS
TRANSFORMATIONAL HELPING US GET
WHERE WE ARE TODAY.
I'LL TELL YOU ONE OTHER AMAZING
STORY THAT'S JUST EVOLVED OVER
THE PAST YEAR, NOT A NERVOUS
SYSTEM DISORDER BUT TYPE 1
DIABETES CONSORTIUM WAS ABLE TO
INNOVATE BY HAVING THE
REGULATORY AGENCIES QUALIFY USE
OF ISLET AUTO ANTIBODIES FOR
PREVENTION, THREE YEARS AGO THAT
HAPPENED.
IN NOVEMBER OF LAST YEAR FIRST
PREVENTION DRUG FOR TYPE 1
DIABETES HAPPENED.
ALL OF THAT HAPPENED THROUGH
BIOLOGIC STAGING OF DISEASE,
UNRAVELING IN ALZHEIMER'S,
PARKINSON'S INITIATIVE THANKS TO
STRONG LEADERSHIP FROM THE
MICHAEL J. FOX FOUNDATION AND
WORLDWIDE LEADERS, OUTLINING
BIOLOGIC STAGING FOR PARKINSON'S
WITH THE FDA.
IT'S JUST INCREDIBLY EXCITING
CHANGES THAT ARE HAPPENING SO
FAST.
>>LOVE TO HEAR THAT.
ALIZA?
>>AS I REFLECT BACK, WHAT
CHANGED IS A MINDSET HOW WE
THINK ABOUT IT, FROM THIS -- AS
MENTIONED, MOVING FROM, OH, THAT
WOULD BE NICE BUT IT'S HARD, TO
HOW ARE WE GOING TO DO IT, WHAT
TOOLS DO WE NEED, WHAT IS THE
OVERSIGHT, WHAT IS THE PROCESS,
WHAT ARE THE STEPS.
REALLY THE FACT THAT WE'RE
HAVING THIS CONVERSATION.
I'M NOT SURE WE WOULD HAVE FIVE
YEARS AGO.
THIS IS INTEGRATED TO EVERY
CONVERSATION ABOUT PROGRESS.
>>AND INDIVIDUALLY THE PROGRESS
THROUGH DATA IS REALLY JUST THE
DIAGNOSTIC HELP IT'S BEEN ABLE
TO GIVE TO ME.
I LOOK PERFECTLY NORMAL BUT
THERE'S A LOT OF ISSUES THAT
WHEN PHYSICIANS HAVE DISMISSED
ME, THE DATA HAS COME BACK TO
SHOW OTHERWISE.
SO I THINK IT'S REALLY IMPORTANT
WHEN WE THINK ABOUT DATA AND HOW
PATIENTS ARE ABLE TO COLLECT AND
USE THEIR DATA IN THEIR OWN
CARE.
SO, GOING FROM THERE,
INNOVATION, GOVERNMENT
INITIATIVE, WE WANTED TO TOUCH
ON WHAT WE'VE SEEN THERE, AS
EACH OF YOUR ORGANIZATIONS, WHAT
HAVE YOU EXPERIENCED ON THAT
SIDE OF IT?
DIANE, WE'LL GO WITH YOU AGAIN.
>>SO GOVERNMENT INITIATIVES
AND, AGAIN, I'LL HIGHLIGHT NINDS
EFFORTS AND FUNDING RESEARCH FOR
SO MANY NOVEL THERAPEUTIC
TARGETS AND INITIATIVES LIKE THE
BRAIN INITIATIVE, INCREDIBLE
ADVANCES IN TECHNOLOGY, NOVEL
TARGETS FOR THERAPY, WE'RE ALL
FOCUSED, A CHAMPION FOR THE FDA,
I DO THINK DIFFERENT
STAKEHOLDERS PARTICULARLY
PATIENT COMMUNITIES DON'T REALLY
UNDERSTAND THE ROLE AND DRIVING
CHANGE THAT THE FDA HAS HAD IN
21ST CENTURY CURES IS JUST ONE
EXAMPLE.
DR. MICHELLE CAMPBELL IS JOINING
THIS CALL, TRULY
TRANSFORMATIONAL EFFORTS ACROSS
NERVOUS SYSTEM DISORDERS BECAUSE
OF THIS RADICAL CHANGE IN THE
PATIENT VOICE.
I SEE IT AFFECTING DRUG
DEVELOPMENT EVERY DAY, AND
PROBABLY THE BEST EXAMPLE I CAN
SAY IS THE CRITICAL PATH
INITIATIVE RECIPIENT OF FUNDS, A
JOINT PARTNERSHIP, CRITICAL PATH
FOR RARE NEURODEGENERATIVE
DISEASES THAT INCLUDES ALS AND
HUNTINGTON'S DISEASE AND FTD,
MANY DISORDERS HIGHLIGHTED AT
THIS MEETING CAME FROM FUNDING,
THE RECOGNITION IF WE'RE GOING
TO BRING TOGETHER WITH A PATIENT
VOICE FRONT AND CENTER, TRULY
TRANSLATIONAL DATA THAT'S GOING
TO ATTACH VALUE AND GET US THESE
THERAPIES AS FAST AS WE CAN WITH
PATIENTS FRONT AND CENTER IS
GOING TO TAKE THE FUNDS TO BRING
THIS DATA FORWARD IN UNIQUE
WAYS.
SO, AGAIN, RECOGNITION OF THE
ROLE OF REGULATORY AGENCIES
CANNOT BE EMPHASIZED ENOUGH.
>>EITHER OF THE TWO OF YOU WANT
TO TAKE THAT FIRST?
>>SURE.
I'M PRIVILEGED TO BE -- NORD --
SORRY, I'LL TRY THAT AGAIN.
SO, NORD ACTUALLY IS WORKING
CURRENTLY NOW WITH THE CRITICAL
PATH INSTITUTE ON A PROJECT
THAT'S FUNDED BY FDA.
THIS IS PART OF THE RARE DISEASE
CURES ACCELERATOR PROGRAM.
WHAT THIS PROGRAM IS, TO CREATE
WHAT WE CALL DATA AND ANALYTICS
PLATFORM.
AND WHAT THIS PROGRAM IS
ENVISIONING IS THIS CENTRAL
REPOSITORY OF DATA, AND IT
CURRENTLY -- THE PLATFORM
EXISTS, THERE'S DATA ON THE
PLATFORM.
AND THE GOAL OF THIS, THIS IS
THE PLACE THAT PEOPLE WILL GO,
THAT REGULATORS WILL GO, THAT
RESEARCHERS WILL GO, THAT
PATIENTS AND FAMILIES CAN GO.
THIS IS WHERE THE RARE DISEASE
DATA WILL SIT AND AS PART OF
THIS PLATFORM ARE THE TOOLS FOR
PEOPLE TO INTERROGATE THE DATA,
TO ASK QUESTIONS OF THE DATA,
AND TO USE THE DATA IN THIS
CENTRAL REPOSITORY.
AND SO TO ME THIS IS REALLY LIKE
A CORE INITIATIVE OF FDA TO
REALLY START TO THINK ABOUT WHAT
IS DATA SHARING REALLY LOOK LIKE
IN A PRACTICAL WAY.
ALREADY TO DATE, THERE'S
CLINICAL TRIAL DATA, THERE'S
SEVEN OF THE NATURAL HISTORY
STUDIES ON THE PLATFORM, THERE
ARE OTHER GROUPS THAT HAVE
CONTRIBUTED DATA.
AND ALL TOGETHER, THIS DATA IS
REALLY HELPING US LEARN AND
INFORM THE DRUG DEVELOPMENT
PROCESS.
>>THAT'S PHENOMENAL.
THE WORK NORD AND C-PATH ARE
DOING ARE SETTING A WONDERFUL
EXAMPLE.
I THINK THE PLACES WHERE I'M
MOST HOPEFUL ARE IN THE FACT
THAT THE FRAMEWORKS THAT WE'RE
SETTING UP ARE DESIGNED WITH
LONG-TERM THINKING IN MIND.
SO WHEN WE THINK ABOUT WHAT ONC
HAS DONE, AS WELL AS FDA IN
TERMS OF GUIDANCE, FROM A
REGULATORY STANDPOINT, THESE
WEREN'T -- WHEN WE THINK ABOUT
INTRAOPERABILITY, THEY WEREN'T
ONE AND DONE.
THEY WERE SET UP AT FRAMEWORKS
WITH CONTINUOUS FEEDBACK LOOP.
WHEN WE THINK ABOUT
INTRAOPERABILITY TO THE POINT
CHRISTINE WAS MAKING, WE'RE AT
THE TIP OF THE ICEBERG WHEN IT
COMES TO WHAT WE CAN ACCESS AND
DO.
BUT THE FRAMEWORKS WERE PUT IN
PLACE AT THE EXACT SAME TIME TO
SAY, HERE IS HOW WE'RE GOING TO
EXPAND THIS YEAR OVER YEAR AND
HAVE A CONVENING MOTION WHERE
FOLKS WHO ARE SPECIALISTS IN A
GIVEN RARE DISEASE OR GIVEN
SPECIALTY CAN BRING THEIR
CONTRIBUTIONS TO THE GENERAL
PUBLIC, WE CAN GET IT INTO THE
NEXT GENERATION AND PUSH IT OUT
EVERY YEAR.
FOR INSTANCE, ONE OF THE THINGS
I'M WORKING ON RIGHT NOW IS FOR
THOSE OF YOU WHOSE TREATMENT
PATHWAYS OR DISEASE DIAGNOSTICS
ARE HEAVILY IMAGING BASED YOU'LL
KNOW THE PATIENT ACCESS RULES
RIGHT NOW REALLY DON'T GIVE
ACCESS TO THE CORE IMAGING DATA.
YOU CAN ONLY GET SUMMARIZED DATA
IN THE EHR.
NOW, THE IRONY, THAT DATA IS
REALLY CLOSE BECAUSE DISTANCE
RADIOLOGY HAS BEEN A THING FOR A
VERY LONG TIME.
WE KNOW HOW TO SHARE
RADIOGRAPHIC IMAGES AND
RADIOLOGY DATA.
BUT IT'S JUST ONE STEP AWAY FROM
THE PATIENT ACCESS.
I'M WORKING ON THE ARGONAUT
PROJECT, IT'S THE GRANDDADDY OF
WHAT GOES INTO THE HL 7 FHIR
SPECIFICATIONS AND BASE CORE
DATA FOR INTERDISCIPLINARY ON.
BUT THERE'S NOW A NEW PROJECT,
SPECIFIC LIVE ON BRINGING
IMAGING DATA INTO THE PATIENT
ACCESS RULE, SO WE'RE PUTTING
TOGETHER WORKING WITH A VARIETY
OF NON-PROFITS WHO NEED IMAGING
IN PATIENT REPOSITORY TO PUT
TOGETHER REFERENCE
IMPLEMENTATION AND DESCRIBE TO
THE WORLD HOW THIS WORKS IN A
WAY THAT'S REPEATABLE AND
CODIFIED.
THAT'S WHAT I'M HOPEFUL FOR, YOU
CAN SEE INNOVATIONS CONTINUING
BECAUSE OF THE FRAMEWORK THE
GOVERNMENT SET UP AND FUNDING
THAT'S NOW THERE, IN SOME CASES
STICKS, NOT JUST CARROTS, TO
MAKE SURE FOLKS FOLLOW ALONG.
>>ONE OTHER INITIATIVE, THE
"ALL OF US" RESEARCH PROGRAM.
SO ONE OF THE THINGS I THINK
ABOUT IS WE ALL HAVE THIS HOPE
TO GET TO PRECISION AND
PERSONALIZED MEDICINE BUT
WITHOUT DATA WE WILL NEVER GET
THERE.
AND SO I THINK THAT'S ONE OF THE
THINGS THAT AS PATIENTS WE HAVE
TO THINK ABOUT, WE HAVE TO SHARE
THAT DATA IN ORDER TO GET TO
DIFFERENT INNOVATIONS FOR
PEOPLE.
BUT YOU MENTIONED
INTRAOPERABILITY.
WHEN WE TALK ABOUT
INTRAOPERABILITY, WHAT DO YOU
GUYS SEE NOW AND WHAT IS YOUR
HOPE FOR THE FUTURE REGARDING
INTRAOPERABILITY?
I DON'T KNOW IF WE WANT TO GO TO
ONE OF YOU OR DIANE.
DIANE, DO YOU WANT TO GO FIRST?
>>SURE, I'LL START, YES.
SO, ONE OF THE INCREDIBLE
REWARDING FACTS ABOUT WORKING AT
C-PATH, WE HAVE AN UNBELIEVABLE
TALENTED GROUP OF DATA
SCIENTISTS THAT SPEND SO MUCH
TIME.
IT'S LIKE DOING A PUZZLE THAT
YOU CAN'T IMAGINE OF 10,000
PIECES TO GO IN AND TRY TO
UNDERSTAND HOW TO HARMONIZE DATA
COLLECTED FROM DIFFERENT STUDIES
AROUND THE WORLD.
EVEN DIFFERENT CLINICAL TRIALS
THAT HAVE THE SAME TARGET
POPULATION HAVE A WHOLE VARIETY
OF DIFFERENT WAYS THAT THEY
COLLECT THE DATA.
AND I THINK FOR ME THE DREAM
WOULD BE TO TRY TO UNDERSTAND
HOW WE CAN GET CONSENSUS UP
FRONT ON DATA STANDARDIZATION.
PEOPLE THAT HAVE WORKED IN THIS
FIELD SO LONG MAYBE DON'T
UNDERSTAND THE REASON WHY ADME
AND PPMI, ALZHEIMER'S DISEASE
AND PROGRESSION MARKER
INITIATIVE WERE SO SUCCESSFUL.
ONE FACTOR IS DATA SHARING THAT
LED TO THE CONCEPT OF BIOLOGIC
STAGING WITH BIOMARKERS, BUT
REALLY THE KEY SUCCESS THERE IS
THE STANDARDIZATION UP FRONT.
THEY AGREED TO COLLECT DATA AND
UNIFIED WAY ACROSS MULTIPLE
SITES AROUND THE WORLD.
AT THE ENDS SO YOU CAN INTEGRATE
AND UNDERSTAND HOW THIS DATA ALL
FITS TOGETHER.
IF THE WORLD WOULD RECOGNIZE
ESPECIALLY INDUSTRY TRIALS
ALIGNING UP FRONT IS GOING TO
HELP EVERYBODY IN THE ENDS,
BECAUSE IN THIS WORLD OF DIGITAL
TECHNOLOGY, JUST THE CONCEPT OF
HARMONIZING DEVICES ACROSS
DIFFERENT PLATFORMS IS
INCREDIBLY DIFFICULT.
I THINK THAT THAT'S JUST NOT
RECOGNIZED YET SO WE WANT TO
AGREE ON CONSENT AND STANDARDS
UP FRONT TO HELP US ALL DOWN THE
ROAD.
>>100% AGREEMENT.
I THINK THE PLACES WHERE --
ACTUALLY A GOOD FRIEND WHO IS AN
EXPERT IN THE USE OF EYE
TRACKING FOR AUTISM EARLY
DIAGNOSIS, AND REALLY GETTING
DOWN TO THAT MICROSECOND
ALIGNMENT IN ORDER TO BRING
THESE PIECES OF DATA TOGETHER
MAKES THE PROBLEMS WE TRY TO
SOLVE SEEM MORE DOABLE.
I THINK WE'RE SEEING A COUPLE
BIG PICKUPS.
IT'S TO THE POINT I WAS MAKING
EARLIER, MAKING ME HOPEFUL.
ONE, FOR THOSE OF US WHO USED TO
HAVE TO DO SITE-BASED RESEARCH
WHERE HALF THE MEETINGS WERE
GETTING EVERYONE TO SET UP
REDCap INSTANCES THE SAME WAY,
THAT'S CHANGING AS WE USE HL7
FHIR, USING THINGS CONSISTENTLY,
ALIGNMENT IS AROUND HOW WE
DEFINE DATA, CLINICAL CONCEPTS,
WE AGREE THAT'S WHAT THIS THING
IS MEASURING AS OPPOSED TO
MAKING SURE WE HAVE PURE
ALIGNMENT FOR MUNGING DATA LATER
ON.
THAT'S A PICKUP.
THE OF WHERE THE HYPE CURVE HAS
GOTTEN PAST THIS, THIS IDEA JUST
BECAUSE WE'VE DEFINED SOMETHING
IN A STANDARD, THAT WE CAN NOW
LOOK AT IT ACROSS THESE DISEASE
AREAS, AND THEY ARE MAGICALLY
GOING TO COME TOGETHER.
SO TO THE POINT DIANE WAS
MAKING, THE ROLE OF DATA
SCIENTISTS ARE NOT GOING AWAY.
I THINK THERE'S A LEVEL OF RIGOR
THAT'S NOW -- IT'S NICE THAT FOR
A LOT OF NON-PROFITS THAT RIGOR
WASN'T ALWAYS REQUIRED.
BECAUSE WE DIDN'T REALIZE IT WAS
MISSING.
BUT NOW WE DO BECAUSE WE CAN
BRING IN THE LOTS OF DIFFERENT
KIND OF MODALITIES OF DATA, AND
ALL OF A SUDDEN YOU NEED THAT
EPI OR MEDICAL CODER OR DATA
SCIENTIST WHO CAN CLOSE THAT
LOOP TO SAY HERE'S WHAT THAT
MEANS, HERE'S WHAT WE
ACTUALLY -- HERE IS HOW THAT
MAPS DOWN TO THE SPECIFIC
RESEARCH QUESTION WE WERE TRYING
TO ANSWER AND WE CAN BE
CONFIDENT WE'RE ANSWERING IT THE
SAME WAY NOW THAT IT'S NO LONGER
INJURIES SITES.
JUST SITES.
YOU DON'T GET TO PICK.
THEY WENT WHERE THEY WENT.
THE IDEA OF RECONCILING
EVERYTHING STARTS TO GO AWAY.
KNOW -- NOW YOU NEED TO RELY ON
STANDARDS TO MAKE SURE THERE'S A
STARTING POINT SO WE CAN AGREE
ON THE LAST MILE.
NOT
>>NOT MUCH TO ADD.
IN ALL THE WORK WE'RE DOING WITH
PATIENT ADVOCACY GROUPS, THE
WORK IS ON THE EDUCATION AROUND
WHY DOES INTRAOPERABILITY
MATTER, BECAUSE SOMETIMES IT
REQUIRES YOU TO NOT HAVE THE
QUESTION THAT MAYBE YOU THINK IS
THE RIGHT ONE.
IT REQUIRES YOU TO MAYBE NOT
CUSTOMIZE YOUR SURVEY, PERHAPS
AS MUCH AS YOU WOULD WANT.
BUT TO START TO BUILD THIS
UNDERSTANDING AND THIS KNOWLEDGE
AROUND WHY WE ARE DOING DATA
STANDARDS AND WHY IT MATTERS AND
HOW IT ULTIMATELY HELPS THE
RESEARCH, SOME SPACES WE'VE
REALLY BEEN THINKING ABOUT.
>>I LOVE ALL OF YOUR RESPONSES.
MOVING FORWARD THERE'S GOING TO
BE A LOT OF BIG BREAKTHROUGHS IN
DATA, CHAT GPT, LARGE A.I. AND
LANGUAGE MODELS.
WHAT UPCOMING BREAKTHROUGHS ARE
YOU ANTICIPATING AND HOW CAN
ORGANIZATIONS PREPARE TO
LEVERAGE SOME OF THESE THINGS?
YOU CAN GO FIRST.
>>I THINK THERE'S TWO VERY --
TWO PARALLEL THINGS THAT ARE
EXCITING, KIND OF COMING
TOGETHER.
ONE IS THE SOURCES OF DATA ARE
CHANGING AND GROWING.
AS WE THINK ABOUT, YOU KNOW --
DATA IS NO LONGER SOMEONE
FILLING OUT A SURVEY.
IT'S THE WATCH ON THEIR HANDS.
IT'S THE PUMP IN THEIR SIDES.
OUR IDEA OF WHAT DATA WE HAVE IS
GROWING EXPONENTIALLY.
AND THE POTENTIAL FOR THAT DATA
IS GROWING.
BUT THEN THAT SORT OF CALLS THE
SECOND PART, WHAT DO WE DO WITH
THESE STREAMS AND STREAMS OF
DATA?
AND I THINK THIS IS WHERE THE
ANALYTICS PIECE THANKFULLY IS
CATCHING UP AND THE MODELS TO
TRY AND TAKE THIS DATA AND TRY
TO MAKE SENSE OF IT TO TRY TO
UNDERSTAND WHAT IT'S TELLING US
SO WE KNOW THE STORIES COMING
FROM THIS DATA.
THOSE TWO THINGS TOGETHER FOR ME
ARE REALLY EXCITING BUT THEN
THERE'S THE QUESTION WHAT DO WE
DO WITH IT?
AND THESE ARE SOME OF THE
CONVERSATIONS WE'VE STARTED TO
HAVE WITH GROUPS, STARTING TO
THINK ABOUT, OKAYF YOU HAVE THIS
STREAM OF GLUCOMETER DATA, HOW
CAN WE DISTILL IT THAT A PATIENT
IS LIKE, OH, WAIT, THERE'S A
PROBLEM, I NEED TO DO SOMETHING,
WHEN A CLINICIAN SEES THIS
INFORMATION, THAT THEY CAN SEE A
SIGNAL AND AS A RESEARCHER
LOOKING A T THIS AGGREGATE DATA
WHAT CAN WE LEARN FROM IT?
>>GREAT RESPONSE.
DAVID, DO YOU WANT TO TAKE IT?
>>YEAH, NOW IT'S SUSPENSEFUL.
SO, I THINK THAT WAS A BRILLIANT
ANSWER.
I THINK THE THEME I'M EXPECTING,
ONE THING I'M THRILLED AND
WORRIED ABOUT, SAME ROOT CAUSE,
THE POINT MADE, THE VOLUME OF
DATA IS EXPLODING, GENERALLY A
GOOD THING.
I THINK IT'S GENERALLY BEING
DIRECTED THROUGH PATHWAYS WE CAN
ACCESS, NOT JUST EXACERBATING
THE IDEA OF HOW THE HECK DO I
INTEGRATE THIS DATA.
IT'S EXPLODING THROUGH
FRAMEWORKS THAT WE CAN TAP INTO.
WHAT WORRIES ME IS TWO THINGS.
SO, ONE, THE DATA THAT WE'RE ALL
COLLECTING RIGHT NOW, WE'RE
ESSENTIALLY JUST -- WE'VE JUMPED
THE SHARK IN TERMS OF WE'RE THE
MAIN CONSUMERS OF THAT DATA.
SO WE'RE BASICALLY BUILDING
TRAINING DATASETS FOR A.I. NOW.
WHEN WE THINK ABOUT REGISTRATION
AND DATASETS, WE'RE ESSENTIALLY
BUILDING TRAIN SETS FOR A.I.
THAT TECHNOLOGY IS ADVANCING
FASTER THAN MOST OF OUR
GOVERNANCE MODELS.
EVEN OUR BUSINESS MODELS CAN
TAKE INTO ACCOUNT.
TWO, THERE'S A WORRY THERE.
THAT IT WILL GET AHEAD OF US,
AND WE'RE ACTUALLY SEEING THIS
IN THE MEDIA AND OTHER THINGS,
FOLKS ARE LIKE, OH, WAIT, YOU
TRAINED YOUR DATA ON ALL THE
MOVIES I WROTE?
NOBODY ASKED ME.
IS IT TOO LATE TO GET THAT BACK?
YES.
NEXT TIME LET'S THINK ABOUT IT.
I DON'T WANT TO SEE THAT HAPPEN
WITH HEALTH DATA.
SECOND, WE'VE GIVEN PATIENTS
TREMENDOUS POWER OVER THEIR DATA
WROUGHT ANY FORM OF EDUCATION TO
THE -- WITHOUT ANY FORM OF
EDUCATION WHAT THAT MEANS, WHO
YOU SHOULD GIVE IT TO AND WHY
YOU SHOULD USE IT, ANOTHER PLACE
THE TECHNOLOGY HAS RUN AHEAD OF
THE ABILITY TO ACCESS THE DATA.
THERE'S SMART FOLKS, SOME TRYING
TO DO THE RIGHT THING, SOME SEE
AN OPPORTUNITY FOR MONEY.
AND THEY ARE GROWING TO -- GOIG
TO THE SAME GROUPS.
THAT GIVES ME PAUSE, I'M NOT
SURE HOW ANY OF US CAN DRIVE
THAT.
IT HAS TO BE A COLLABORATIVE
EFFORT, PROBABLY A CONGRESSIONAL
EFFORT TO PUMP THE BRAKES A
LITTLE BIT.
BUT ENDING ON A POSITIVE NOTE, I
DO THINK THAT THE A.I.
APPROACHES ARE GOING TO ALLOW US
TO DO THINGS THAT WERE SIMPLY
IMPOSSIBLE BECAUSE OF THE VOLUME
OF DATA, LIKE THERE WEREN'T
ENOUGH DATA SCIENTISTS.
SO THE ABILITY TO BE ABLE TO
START AGGREGATING AND
INTEGRATING INFORMATION,
ANSWERING QUESTIONS, THAT ALLOWS
SOMEONE BEING SEEN IN A RURAL
HEALTH SETTING, WHO HAS A RARE
DISEASE, THAT DOCTOR IS NEVER
GOING TO SEE ANOTHER PATIENT
LIKE THAT AGAIN TO GET A NUDGE
FROM AI, THIS IS WHAT YOU'RE
SUPPOSED TO DO WHEN SOMEONE
PRESENTS, A LEVELING EFFECT
BECAUSE A.I. TOOLS ARE NOT
EXPENSIVE TO DEPLOY.
I SEE THAT AS POSITIVE.
ENDING ON A NEGATIVE, THE FLIP
SIDE IT'S GOING EXACERBATE
DIVERSITY, EQUITY, AND INCLUSION
CHALLENGES THAT WE ALREADY HAVE.
BECAUSE SO MANY OF THESE TOOLS
ARE BASED ON DATA THAT ARE
DIGITALLY GENERATED, THAT
REQUIRE EXPENSIVE CARE SETTINGS
TO GENERATE THEM IN THE FIRST
PLACE, EXPENSIVE SMARTPHONES OR
ACCESS TO BROADBAND INTERNET.
WE KNOW THE MAJORITY OF OUR
COUNTRY DOESN'T HAVE ACCESS TO
THOSE THINGS.
IT'S NOT 50/50, IT'S THE
MAJORITY THAT DOESN'T HAVE
ACCESS ON A RELIABLE BASIS, SO I
THINK WE'RE GOING TO SEE
TREMENDOUS BREAKTHROUGHS BUT
PROBABLY A GENERATION OR TWO OUT
BEFORE IT'S MORE EVENLY
DISTRIBUTED.
>>DIANE?
>>I HAVE A BIPOLAR VIEW, ON THE
ENTHUSIASTIC SIDE POST-COVID,
WITNESSING THE CHANGES IN
DECENTRALIZED TRIALS, DIVERSITY,
YOU KNOW, WE'RE FINALLY GOING TO
BE IN A POSITION TO REACH PEOPLE
TO GO INTO TRIALS THAT
HISTORICALLY, EVEN PEOPLE IN
RURAL SETTINGS, IT'S HAPPENING,
RIGHT?
SO THIS IS A JUNCTURE WHERE BOTH
MY HAT AS A NEUROSCIENTIST IS
CONFOUNDED WITH THE EXPERIENCE I
HAD IN WITNESSING, MY BROTHER
DIED OF PARKINSON'S AT AGE 59
LAST YEAR.
HE WAS A COMPUTER SCIENTIST, BUT
THERE WAS NO IMPLEMENTATION OF
HOW DIFFERENT STAKEHOLDERS IN
HIS ENVIRONMENT COULD
COMMUNICATE TOGETHER.
THE REALITY IN TODAY'S SETTING
DESPITE THE HYPE AND INCREDIBLE
PROGRESS IN TECHNOLOGY, THE
REALITY OF WHAT HAS TO HAPPEN
WITH PATIENTS AND FAMILIES ISN'T
THERE YET, RIGHT?
AND WHAT WE DO AT C-PATH FOR
EVERYTHING WE DO TO BE
SUCCESSFUL AT ACHIEVING
REGULATORY ENDORSED DRUG
DEVELOPMENT TOOLS, BIOMARKERS,
OUTCOME MEASURES, TOOLS ARE
POWERFUL BUT WE NEED TO SHARE
DATA.
THE PARKINSON'S CONSORTIUM,
LEADING A PROJECT FOR SMART
PHONES TO DEVELOP WAYS TO DETECT
AND MONITOR PROGRESS IN EARLY
STAGE DISEASE, THAT DATA IS
BEING USED TO HELP INFORM
PATIENTS.
FACETS THEY HAVE NEVER
UNDERSTOOD, WE'RE MAKING GREAT
PROGRESS, PATIENTS ARE DRIVING
THAT CHANGE.
BUT ON THE OTHER SIDE, INHERENT
CHALLENGES OF THE ENTIRE FIELD,
FOR WHATEVER BUSINESS REASONS,
LEGAL, FINANCIAL, WHATEVER, THE
ABILITY TO SHARE THIS DATA IS
INCREDIBLY DIFFICULT.
BRINGING IN THE TECH PROVIDERS,
HOW TO GET EVERYBODY ON THE SAME
PAGE, TO SHARE NOT JUST THE
WINNING BUT THE CHALLENGES
BECAUSE THE ISSUES YOU RAISE
ABOUT INTRAOPERABILITY, HOW DO
WE HARMONIZE AND WORK WITH
VOLUMES OF DATA, WE'RE STILL NOT
SHARING THAT YET, THAT'S SLOWING
PROGRESS.
IF WE COULD HELP, AGAIN, UTILIZE
THE PATIENT VOICE TO DRIVE
CHANGE FOR INNOVATION AND SHARE
THE LEARNINGS, AND SUCCESSES AND
FAILURES, WE'LL MAKE INCREDIBLY
FAST PROGRESS ACROSS ALL OF
THESE DISEASES THAT NEED THESE
TOOLS.
>>CAN YOU KEEP ME GOOD ON TIME?
TWO CLOCKS HAVE TWO TIMES.
FIRST I WANT TO SAY THAT A.I. IS
JUST A TOOL.
AND THAT WE HAVE TO LOOK AT THAT
WAY.
IT'S NOT AN END END-ALL, BE-AL,
IT'S GOING TO HELP US IMPROVE
THE PROCESS BUT WILL NOT TAKE
OVER.
AND THE SECOND THING IS
EDUCATION AND AWARENESS TO
PATIENTS AROUND THE DATA.
AS WE MOVE INTO THIS DIGITAL
WORLD AND DECENTRALIZE CLINICAL
TRIALS, PATIENTS ARE GOING TO
HAVE TO LEARN HOW TO COLLECT
DATA, KNOW WHY THEY ARE
COLLECTING IT AND IT HAS TO BE
CLINICAL GRADE DATA TO WORK WITH
IT.
WORKING WITH PATIENTS TO
ESTABLISH THOSE THINGS IS GOING
TO BE EXTREMELY IMPORTANT.
AND WHEN THEY KNOW HOW AND WHY
THEY WILL TRUST SHARING MORE OF
THE DATA, SO THAT'S ONE OF THE
THINGS WE SHOULD THINK ABOUT.
BUT MOVING ON WHEN TALKING ABOUT
SHARING DATA, WHAT DO YOU THINK
THE CORE PRINCIPLES SHOULD --
WHAT CORE PRINCIPLES SHOULD
GUIDE THIS DATA SHARING?
I HAVE MY OWN OPINIONS BUT I'D
LOVE TO HEAR FROM THE DIFFERENT
ORGANIZATIONS HERE.
ALIZA, DO YOU WANT TO GO FIRST?
>>SURE, I LOVE THAT WE TALK
ABOUT --
>>MAKING IT FUN.
>>I LOVE THAT WE'RE TALKING
ABOUT GUIDING PRINCIPLES AROUND
DATA SHARING, THERE'S LIKE THIS
ETHOS, THIS APPROACH.
I THINK THAT'S SO TELLING OF
SORT OF HOW WE'RE THINKING ABOUT
THIS.
AND I WAS RECENTLY ACTUALLY
TALKING WITH ONE OF THESE GROUPS
STARTING TO BUILD THEIR DATA
GOVERNANCE, AND THAT WAS WHERE
WE STARTED.
WHAT ARE THE GUIDING PRINCIPLES?
HOW ARE WE COMING INTO THIS?
ARE WE COMING INTO THIS WITH AN
IDEA THAT THE GOAL HERE IS
SHARING IS OUR GOAL AND WE WANT
TO DO WHAT'S POSSIBLE?
BUT THEN WHERE DO WE NEED TO BE
CAREFUL?
WHAT DO WE NEED TO BE THINKING
ABOUT?
ESPECIALLY IN THIS RARE DISEASE
SPACE, NUMBERS ARE SMALL.
PRIVACY BECOMES A TRUE CONCERN.
SO HOW DO WE INTEGRATE THAT INTO
HOW WE'RE THINKING ABOUT DATA
SHARING?
>>THAT'S A PHENOMENAL POINT.
I THINK THE TENSION, AND I'VE
SEEN THIS ON BOTH SIDES, SO FOR
THE EARLY PART OF MY CAREER I
WAS SUPPORTING ACADEMIC RESEARCH
IN NEURODEVELOPMENTAL DISORDERS,
AND I WILL NEVER FORGET A PARENT
PULLING ME ASIDE WHEN I WAS
ASKING A QUESTION ABOUT CONSENT.
AND THEY SAID, I DON'T EVEN
UNDERSTAND WHY WE DO THIS, IF
YOU TOLD ME THAT THE RESEARCH
FOR MY CHILD WAS ONLY GOING TO
BE LOOKED AT BY FOUR SMART
PEOPLE IN THAT ROOM I WOULD SAY
FROM A DATA SHARING PERSPECTIVE
I WOULD SAY I'M NOT INTERESTED.
I WANT EVERY SMART PERSON WHO
CAN POTENTIALLY HELP MY CHILD TO
LOOK AT THIS AND MOVE IT
FORWARD.
AND THAT WILL ALWAYS STAY WITH
ME AS THE NATURAL TENSION
BETWEEN PRIVACY AND THE
WILLINGNESS FOR A LOT OF
FAMILIES TO SAY I DON'T WANT
ARTIFICIAL RESTRICTION BECAUSE I
FORGOT TO CHECK A BOX OR
SOMETHING WASN'T EXPLAINED, NOW
SMART PEOPLE CAN'T TRY TO
ADVANCE THIS FIELD.
OTHER FOLKS ARE AT THE OTHER END
OF THE SPECTRUM, SAYING I WANT
TO MAKE SURE IT'S ONLY SEEN BY
ONE PERSON FOR THIS ONE THING.
I THINK THE REQUIREMENTS FOR US
TO MAKE ADVANCES ARE GOING TO BE
SOMETHING THAT LOOKS MORE LIKE
BROAD CONSENT.
FROM AN EDUCATION PERSPECTIVE,
IT'S REALLY ON THE ONUS OF ALL
OF US TO EXPLAIN, YOU KNOW, HOW
IS IT NOT SCARY, HOW ARE THE
GOVERNANCE MODELS IN PLACE,
BECAUSE DATA FLUIDITY IS SO
GREAT, BARRIERS HAVE BEEN
REDUCED.
LOOK AT THE WORK C-PATH IS DOING
WITH DATA THAT WAS LOCKED AWAY
FOR A VERY LONG TIME.
YES, THERE'S A CHALLENGE
BRINGING THAT TOGETHER.
YES, THAT'S PRIMARILY
DE-IDENTIFIED DATA SO YOU'RE NOT
GOING BACK TO FOLKS BUT THAT
MODEL IS GOING TO FLIP TO WHERE
WE'RE GOING TO BE SITTING ON
LARGE REPOSITORIES OF IDENTIFIED
PATIENT DATA THAT WAS CONSENTED
FOR ONE REASON AND IMPOSSIBLE TO
GO BACK AND CONSENT THEM FOR
ANOTHER VALID REASON.
SO I THINK THAT TO ME IS REALLY
WHERE THE CHALLENGE IS GOING TO
BE IN TERMS OF WORKING WITH
PARENTS AND PATIENTS TO SAY,
THIS IS WHY WE'RE ASKING FOR
MANY A BROADER PURVIEW ON WHO
MIGHT BE ABLE TO CONTRIBUTE TO
YOUR CARE OR TO THIS RESEARCH.
AND LET'S HAVE THAT QUESTION UP
FRONT BECAUSE WE DON'T GET
ANOTHER CHANCE TO HAVE THAT
CONVERSATION ON.
WE'VE SEEN SO MANY AMAZING
OPPORTUNITIES FROM LINKING DATA
OR BRINGING IT FORWARD, AND NOW
THE PRIMARY REASON WE CAN'T DO
IT BECAUSE WE DIDN'T THINK ABOUT
CONSENTING FOR THAT AND CAN'T GO
BACK AND FIND THESE FOLKS.
>>I THINK DYNAMIC CONSENT IS
SOMETHING WE NEED TO LOOK INTO.
WE MAY TACKLE THAT IF WE HAVE
TIME.
DIANE, WHAT ARE YOUR THOUGHTS ON
THE QUESTION?
>>YES, ECHOING THE IMPORTANCE
OF CONSENT, ONE OF THE
CHALLENGES AGAIN WE'VE HAD OVER
THE YEARS AT C MATH IS THE
NEGOTIATION OF RECONSENTING.
PEOPLE DON'T UNDERSTAND HOW
CHALLENGING THAT IS, ALMOST
IMPOSSIBLE, RIGHT?
CONGRATULATIONS TO PENNY DACKS
WHO IS THERE WHO WORKS WITH US
IN THE ALZHEIMER'S CONSORTIUM
YEARS AGO TO DEVELOP A
PUBLICATION ON SOME TYPE OF
UNIVERSAL CONSENT THAT COULD BE
UTILIZED FOR DEMENTIA STUDIES,
RIGHT?
BECAUSE THE LANGUAGE IS REALLY
CRITICAL AND PEOPLE TELL ME THAT
THE TYPE OF CONSENT THAT IS
STILL USED TODAY FOR MANY TRIALS
IS VERY DIFFICULT TO UNDERSTAND.
LET ME MAKE ONE POINT.
PARTICULARLY FOR THIS MEETING
AND FORUM, I WISH I WAS THERE,
THE ROLE OF NON-PROFITS IN
DRIVING CHANGE IS NOT
APPRECIATED ENOUGH.
WE WORK WITH EIGHT NON-PROFIT
ORGANIZATIONS FROM AROUND THE
WORLD IN PARKINSON'S, AND
TRANSFORMATION IS HAPPENING
THANKS TO PARTNERS LIKE THE
MICHAEL J. FOX FOUNDATION.
IN THE FUNDS THEY ARE SO
GENEROUS TO PROVIDE TO
RESEARCHERS, IT'S MORE AND MORE
COMMON NOW THAT IN ORDER TO
RECEIVE AND AGREE TO RECEIVE
FUNDING, THAT ONE HAS TO AGREE
TO SHARE THE DATA, RIGHT?
AND TO HAVE THE CONSENT WRITTEN
IN WAYS THAT UP FRONT YOU'RE NOT
GOING TO HAVE TO GO BACK AND
WORRY ABOUT RECONSENT BECAUSE
ALL OF THAT IS AGREED UPON UP
FRONT.
CONDITIONAL ON THE FUNDING,
RIGHT?
JUST LIKE NIH IS DOING AROUND
OPEN DATA SHARING.
THIS IS THE WORLD THAT'S GOING
TO HAPPEN GOING FORWARD.
WE HAVE TO FIGURE OUT GOVERNANCE
MODELS AND PRIVACY AND POLICY
RULES THAT WOULD HELP AVOID
MONETIZING DATA OR CONCERNS THAT
PEOPLE HAVE, OBVIOUSLY, AROUND
PATIENT PRIVACY.
I THINK CHANGE IS HERE BUT
CANNOT EMPHASIZE HOW IMPORTANT
IT IS FOR NON-PROFITS TO BE
FULLY ENGAGED DRIVING THIS
CHANGE.
>>AND I THINK IT'S EXTREMELY
IMPORTANT FOR NON-PROFITS NOWED
--
NOW TO EDUCATE THEMSELVES AND
LOOK AT BUSINESS MODEL OF HEALTH
CARE AND HOW THAT WORKS.
WE HAVE SEVEN MINUTES LEFT.
WE'RE GOING TO CUT THIS DOWN.
ONE THING I WOULD LIKE TO SAY,
YOU KNOW, SHARING THE BENEFITS
WITH PATIENTS.
RIGHT NOW WE HAVE PEOPLE
PARTICIPATING IN CLINICAL
TRIALS.
I'VE OFTEN SAID IF WE COULD
COLLECT DATA FROM TRIALS AND
STUDIES I'VE PARTICIPATED IN WE
WOULD HAVE A HUGE AMAZING
HOLISTIC VIEW OF MYSELF.
BUT THAT'S NOT FULLY SHARED AT
THIS MOMENT.
SO, WHAT ARE YOUR THOUGHTS AND
WHERE DO YOU SEE THIS GOING ON
SHARING SOME OF THESE ACTUAL
RESULTSES OR REPORTS WITH
PATIENTS, OR THE DATA?
>>I CAN LEAD THIS ONE OFF.
I DO A LOT OF THIS NOW IN MY DAY
JOB.
I THINK WHEN WE USED TO PLAN OUT
PATIENT REGISTRIES AND PATIENT
DATA INITIATIVES, WE WOULD SPEND
A LOT OF TIME TALKING ABOUT WHAT
ARE THE SPECIFIC DATA ELEMENTS
THAT WE WANTED TO CAPTURE.
THAT'S STILL PART BUT NOW THE
MAJORITY OF PLANNING GOES INTO
WHAT IS THE GIVE-BACK TO THE
PARTICIPANT?
IF WE'RE GOING TO ASK FOR ALL
THIS INFORMATION CAN WE
SUMMARIZE IT?
CAN WE CREATE AN EMERGENCY PANEL
THAT SAYS I HAVE A RARE DISEASE,
AND YOU NEED TO KNOW THIS ABOUT
ME AND MAKE SURE THAT CAN BE
PRINTED.
EVEN SIMPLE THINGS LIKE THAT
THAT PROVIDE REAL TANGIBLE
BENEFIT BACK TO THE PARTICIPANT
AS A WAY TO SAY THANK YOU FOR
SHARING THIS INFORMATION WITH
ME, HERE'S WHAT I WAS ABLE TO
LOOK AT IT.
THE RESEARCH FOUNDATION DOES A
GLORIOUS JOB WITH THEIR -- WITH
THEIR INITIATIVE IN TERMS OF --
SUSAN G. KOMEN AS WELL, TO WHERE
THEY ARE BRINGING TOGETHER YOUR
CLINICAL RECORDS, RUNNING A
NOVEL ASSAY ON YOU.
THEY ARE GETTING
PATIENT-REPORTED DATA.
IN THE DISPARATE POOLS, THERE'S
SOME INTERESTING STUFF THERE BUT
IT'S DIFFICULT FOR THE
PARTICIPANT TO UNDERSTAND WHAT
THE HECK IS ALL THIS FOR.
AND IT WAS -- PART OF THAT
PROTOCOL TO MAKE SURE WE
SUMMARIZE WHAT WE'VE LEARNED TO
HAVE A BETTER CONVERSATION WITH
THE CARE PEOPLE.
WE DID THIS CLIA-APPROVED TEST,
LEARNED FROM THE PATIENT, TALKED
ABOUT THIS FROM THE MEDICAL
RECORDS, THIS SHOULD BE WHAT YOU
DESCRIBES, WHAT YOU SHOULD BE
DISCUSSING NEXT TIME.
THAT SHIFT IS NOW PERSISTENT,
AND THAT'S -- IT IS ALSO WHAT
MAKES ME WORRIED ABOUT FOLKS
DANGLING, GIVE ME YOUR DATA AND
I'LL SUMMARIZE.
TAKE A LEADERSHIP ON THAT.
>>I WOULD LOVE TO SEE THAT
INFORMATION GO BACK TO MY FIRST
TEAM BEFORE IT COMES BACK TO ME.
ALIZ SARKS, -- ALISA, DO YOU HE
ANY COMMENTS?
>>SURE.
ONE THING I LOVE ABOUT THE WAY
THAT THE PLATFORM WORKS IS IN
REAL TIME, AS SOON AS YOU ENTER
DATA, NOT ONLY ARE YOU ABLE TO
SEE YOUR DATA BUT YOU SEE YOUR
DATA AND EVERYBODY ELSE'S.
SO IT GIVES YOU A SENSE TO
REALLY START TO UNDERSTAND HOW
YOU FIT INTO THE WIDER GROUP OF
PEOPLE WHO ARE CONTRIBUTING AND
TO SEE YOUR SPACE, TO SEE HOW
YOUR DATA IS CONTRIBUTING TO
THAT STORY.
>>AND DIANA, LAST COMMENT FROM
YOU?
>>YES, I'LL BE BRIEF.
TWO WEEKS AGO AT THE WORLD
PARKINSON'S CONGRESS IN
BARCELONA, I WAS UNABLE TO
TRAVEL THIS YEAR BUT ONE OF THE
MOST UNIQUE FORUMS IS BRINGING
PATIENT COMMUNITY TOGETHER
EXPERT RESEARCHERS FROM AROUND
THE WORLD.
IN THAT FORUM THERE IS A WAY TO
SHARE INFORMATION IN WAYS THAT
HAVE BEEN TRADITIONALLY
DIFFICULT AND NEED
INFRASTRUCTURES TO ALLOW HELPING
THE PATIENTS UNDERSTAND WHAT IS
BEING COLLECTED AND HOW TO
INTERPRET THIS KIND OF DATA, AND
FOR ME AS WE CONTINUE TO WATCH
WHAT HAPPENS IN TRANSFORMING
THERAPEUTIC DEVELOPMENT FOR
APPROVALS OF DRUGS THAT ARE
DISEASE MODIFYING, WE NEED TO
EDUCATE ON RESILIENCE, WHAT THEY
CAN DO IN THEIR DAILY LIVES TO
HELP IMPROVE THE QUALITY OF
THEIR LIVES NOW WHILE THERAPIES
ARE UNDER DEVELOPMENT AND SHARED
IN DIFFERENT WAYS.
PATIENT VOICE DATA IS STILL
SOMEWHAT NOVEL, HOW DO WE SHARE
THAT AND BRING THAT TOGETHER
ACROSS DIFFERENT GROUPS DOING
THIS.
IT'S HAPPENING NOW.
I CONGRATULATE MY COLLEAGUES AT
THE MICHAEL J. FOX FOUNDATION,
TRANSFORMING HOW TO BRING THIS
INFORMATION TOGETHER IN
PARKINSON'S FROM AROUND THE
WORLD.
THE TIMING IS NOW.
REALLY FANTASTIC FORUM TODAY TO
HEAR THESE TOPICS DISCUSSED IN
SUCH A DYNAMIC WAY.
THANKS.
>>I WANT TO THANK MY PANELISTS
FOR BEING HERE AND MY THOUGHTS
ON THIS REALLY, ONE OF THE
ORGANIZATIONS I WORK WITH, OUR
SLOGAN IS NO AGGREGATION WITHOUT
REPRESENTATION.
THAT'S IN REGARD TO THE DATA.
WE HAVE ALSO COME UP WITH SEVEN
COLLECTIVE GUIDELINES, SEVEN
GUIDELINES TO COLLECTIVE DIGITAL
RIGHTS.
IF YOU GO TO
LIKECOLLECTIVE.ORG/TRUST WE'RE
WORKING WITH TECH COMPANIES, HOW
TO WE USE THE DATA AND MONITOR
IN THE MEANTIME.
IT'S A HUGE TOPIC.
WE DIDN'T GET TO OUR CONSENT AND
OTHER THINGS.
I DON'T KNOW IF WE HAVE TIME FOR
QUESTIONS.
>>WE HAVE TIME FOR A COUPLE
QUESTIONS.
>>IF ANYONE DOES.
I SEE ONE OVER THERE.
WE'VE GOT BOTH SIDES.
>>I'M SO EXCITED ABOUT WHAT ALL
OF THIS IS GOING TO MEAN FOR THE
FIELD OF BIOMEDICAL RESEARCH.
I ALSO WORRY WE DON'T ALWAYS
TALK ABOUT THE RISKS.
WE TALK ABOUT RISK OF PRIVACY IN
THE FIELD OF DEMENTIA, ONE OF
THE THINGS WE'VE HEARD OF IS
PEOPLE WHO GET MAYBE -- A FAMILY
WHO IS OPEN ABOUT THE GENETIC
RISK OF DEMENTIA IN THEIR FAMILY
CAN SOMETIMES LOSE THEIR JOB.
WE TALK ABOUT TRYING TO GET INTO
UNDERSERVED POPULATIONS AND EARN
THEIR TRUST BUT I DON'T HEAR AS
MUCH CONVERSATION HOW DO WE
DESERVE THEIR TRUST.
ONE SPECIFIC EXAMPLE AS WE
CREATE ALGORITHMS AROUND HOW TO
PRE-
PREDICT WHO IS GOING TO DEVELOP
DEMENTIA TO FIND THEM EARLY, HOW
DO WE MAKE SURE INSURANCE
COMPANIES ARE NOT BLOCKING
INSURANCE OF LONG-TERM CARE AND
HEALTH INSURANCE AND DISABILITY
INSURANCE FOR PEOPLE WHO DON'T
EVEN KNOW THAT THEY ARE AT RISK
OF DEMENTIA, AND SO FROM YOUR
PERSPECTIVES THINKING ABOUT
PATIENT DATA, STEP ASIDE FROM
BENEFITS, WHAT ARE THE RISKS AND
POLICIES IN PLACE TO GUARD
AGAINST THOSE RISKS?
>>I HAVE A TON OF RISK THAT WE
CAN DISCUSS.
I DON'T KNOW ABOUT YOU.
EVEN JUST THE RISK A THIRD
PARTY, HHS ISSUES A BULLETIN
ABOUT REQUIRING PIXELS BE
REMOVED FROM HOSPITAL SYSTEMS,
THERE ARE A LOT OF RISKS.
I DON'T SAY THIS PUBLICLY AT A
LOT OF PLACES BUT I HAVE A
BLUETOOTH-ENABLED DEVICE,
THREATS ON IMPLANTED HAVEN'T
BEEN DISCUSSED YET WITH PATIENTS
EITHER.
A LOT OF THINGS WITH DATA BEING
COLLECTED, THERE ARE A LOT OF
BAD THINGS OUT THERE.
I THINK THINGS LIKE DYNAMIC
CONSENT, LIMITED LICENSING
PARTNERSHIPS, HAVING PATIENTS ON
YOUR BOARDS ARE ALL THINGS THAT
CAN MITIGATE THOSE THINGS AS WE
MOVE FORWARD.
BUT I'D LOVE TO HEAR FROM ALL OF
YOU.
>>YEAH, I'LL JUMP IN THE HOT
SEAT NEXT.
I THINK THERE'S A COUPLE PLACES
WHERE POLICIES ARE REQUIRED, SO
I THINK YOU ALREADY MENTIONED A
COUPLE.
WITH THE EXPOSURE THAT WE'RE
GOING TO HAVE GOING FORWARD, AND
WE'RE WORKING ON SOME OF THE
ITEMS TO HELP WITH EARLY
DIAGNOSIS AND DIGITAL
BIOMARKERS, I DO THINK ABOUT
THOSE THINGS.
OKAY, WHO CAN ACCESS THAT
INFORMATION AND THERE'S NOTHING
PROTECTING PEOPLE FROM THAT
INFORMATION, YOU'RE NOT IN A
PROTECTED CLASS IF WE IDENTIFY
DIGITAL BIOMARKER ON YOUR PHONE.
AND ANYBODY CAN BASICALLY BUY
THAT DATA IN A LOT OF CASES.
SO I THINK THE WORK YOU'RE DOING
WITH TECH COMPANIES, I THINK IT
NEEDS TO GO BEYONDS A BILL OF
RIGHTS FROM A TECH PERSPECTIVE.
I THINK IT REQUIRES
CONGRESSIONAL LEADERSHIP TO SAY
AS WE HAVE NO FORMS OF DATA
BECOMING AVAILABLE THIS IS WHAT
THE DIGITAL RIGHTS ARE RELATED
TO THAT AND EXPAND ON WHAT WE
DID UNDER OBAMA CARE AND OTHER
THINGS, HOW DO WE CLASSIFY, IS
IT PREEXISTING YOU CAN NO LONGER
QUALIFY FOR, SOMETHING THAT
NEEDS TO BE PROTECTED FROM A
POOLING PERSPECTIVE?
RIGHT NOW THERE ISN'T ANY REAL
PROTECTION FOR IT.
YOUR EXAMPLE ALL THE TIME, FOLKS
SAY JUST SHARE, WELL, YOU'RE
USED TO DISEASE AREAS WHERE
SHARING OUTWEIGHS THE RISK.
AND THERE ARE FORMS OF MORE
ADULT ILLNESSES WHERE SOMETIMES
THE SHARING DOES NOT OUTWEIGH
RISK OF RAMIFICATIONS, THERE ARE
NO PROTECTIONS RIGHT NOW FOR IT.
>>DIANE, ANYTHING?
>>YEAH, OTHER THAN PENNY HIT ON
EXACTLY WHAT KEEPS ME UP, THE
WHOLE THING ABOUT INSURANCE.
FOR CRITICAL PATH INSTITUTE
EVERYTHING IS DE-IDENTIFIED
BEFORE IT COMES IN.
WE NEED TO DO BETTER EDUCATING
RISK/REWARD PIECE ESPECIALLY TO
ALL STAKEHOLDERS INCLUDING
PHYSICIANS AND GENERAL
PRACTITIONERS, BECAUSE THE TERM
OF PRIVACY IS MISUNDERSTOOD.
IT'S A WHOLE OTHER TOPIC.
>>THE RISK AROUND SOCIAL MEDIA
SHARING ARE SOMETHING THAT NEED
TO BE DISCUSSED WITH OUR
COMMUNITIES AS WELL BUT WE DON'T
HAVE TIME.
>>A BRIEF BUT ENTHUSIASTIC
ROUND OF APPLAUSE FROM ME.
ONE USER REPORT FROM THE FIELD,
I WANT TO SINGLE OUT IN
PARTICULAR THE WONDERFUL PROGRAM
THAT'S FUNDED BY THE FDA, RUN BY
CRITICAL PATH INSTITUTE, IN
COLLABORATION WITH NORD, THE ONE
THAT ALIZA SPOKE ABOUT, RARE
DISEASE CURES ACCELERATOR AND
DATA AND ANALYTICS PLATFORM,
WE'VE HEARD ABOUT PATIENT DATA,
IMPORTANCE OF NATURAL HISTORY.
WE'VE BEEN WORKING FOR 20 YEARS
ON OUR NATURAL HISTORY DATABASE.
TERRIBLY IMPORTANT, IMPORTANT
FOR PLANNING CLINICAL TRIALS,
FOR RECRUITING OUR CLINICAL
TRIALS, DEVELOPING ENDPOINTS IN
BIOMARKERS.
BUT WHEN THE CRITICAL PATH
INSTITUTE WAS FUNDED BY THE FDA,
FOR THIS RARE DISEASE CURES
ACCELERATOR DATA ANALYTICS
PLATFORM WE SUBMITTED OUR DATA.
WE'VE BEEN BUILDING FOR 20
YEARS.
BUT EACH TIME WE WENT TO THE FDA
WE HAD TO TELL THEM, OKAY, WE
USE NATURAL HISTORY DATA THIS
WAY TO DESIGN THIS.
NOW SINCE WE'RE LEADING DISEASE
GROUP ON THAT PLATFORM, WE'VE
HAD SEVERAL MEETINGS WITH FDA
REVIEWERS AND STATISTICIANS AND
SO FORTH.
THEY HAVE BECOME SO FAMILIAR
WITH OUR NATURAL HISTORY
DATABASE, THEY BUILT THEIR
CONFIDENCE IN A HIGH QUALITY OF
THAT DATABASE AND RELIABILITY OF
THAT DATABASE SO WHEN THIS DRUG
COMPANY REATA LAST SUMMER
SUBMITTED DATASETS BASED ON OUR
NATURAL HISTORY STUDY TO
SUPPLEMENT DRUG APPLICATION THE
FDA SAID THEY NO LONGER FELT THE
NEED FOR ADVISORY COMMITTEE
MEETING AND WERE GOING TO STICK
WITH THE PDUFA DATE OF FEBRUARY
28, RARE DISEASE DAY.
NOW BECAUSE OF THOSE ADDITIONAL
DATASETS BASED ON THIS PLATFORM,
THEY HAVE BECOME FAMILIAR WITH,
THEY ANNOUNCED OUR FIRST
APPROVAL.
SO IT'S TERRIBLY IMPORTANT.
I'LL CONCLUDE BY SAYING IF YOU
DON'T HAVE NATURAL HISTORY DATA,
GET IT.
IF YOU HAVE NATURAL HISTORY DATA
AND IT'S NOT YET ON THE RARE
DISEASE CURES ACCELERATOR
ANALYTICS PLATFORM PLEASE GET IT
THERE.
I WOULD DO YOU TREMENDOUS --
SHOW TREMENDOUS BENEFIT.
>>WE'RE ALL SET.
>>I AM NAOMI BOOKER, HEALTH
PROGRAM SPECIALIST AT NINDS.
OFFICE OF GLOBAL HEALTH AND
HEALTH DISPARITIES WITHIN THE
DIVISION OF CLINICAL RESEARCH.
I'M EXCITED TO PROVIDE A BRIEF
OVERVIEW OF THE HEALTH EQUALITY
STRATEGIC PLANNING OFFICE.
THE NINDS IS DEDICATED TO
ADDRESSING NEUROLOGICAL HEALTH
INEQUITIES, FACED BY VULNERABLE
AND MARGINALIZED POPULATIONS.
AS WE WORK TO CONTINUE THE
NEUROLOGICAL HEALTH FOR ALL
PEOPLE.
DESPITE THIS, THERE'S STILL
POPULATIONS THAT EXPERIENCE
HEALTH DISPARITIES.
AS A RESULT AS YOU CAN SEE ON
THE SLIDE WE HIGHLIGHT THE
CURRENT NIH DEFINED POPULATIONS
THAT EXPERIENCE HEALTH
DISPARITIES.
THE NEUROLOGICAL HEALTH
CHALLENGES OF PEOPLE WITH LIVED
EXPERIENCES MAY COMPOUND WHEN
THEY IDENTIFY AT THE
INTERSECTION OF MORE THAN ONE
MARGINALIZED GROUP.
TO WORK FROM A SHARED
UNDERSTANDING OF HEALTH
DISPARITIES, WE USE HEALTHY
PEOPLE 2030 HEALTH DISPARITIES
DEFINITION.
HEALTH DISPARITIES IS NOT SIMPLY
A DIFFERENCE IN HEALTH OUTCOMES
BUT A DIFFERENCE TIED TO
POPULATIONS EXPERIENCING GREATER
OBSTACLES BASED ON CHARACTERS
LINKED TO DISCRIMINATION OR
EXCLUSION.
THE GOAL OF THE NINDS STRATEGIC
PLANNING EFFORT IS TO ADVANCE
HEALTH EQUITIES THROUGH
NEUROLOGICAL RESEARCH.
AND TRAINING AND NOTE EMPHASIS
ON EQUALITY AND EQUITY.
IN HEALTH EQUITY INDIVIDUALS ARE
EMPOWERED TO ATTAIN HIGHEST
OPTIMAL HEALTH, THERE'S NO
ONE-SIZE-FITS-ALL APPROACH TO
HEALTH OPPORTUNITY AND UNIQUE
DIFFERENCES ACROSS POPULATIONS
MUST BE CONSIDERED.
IN 2020, THE NINDS EMBARKED ON A
TWO-YEAR STRATEGIC PLANNING
EFFORT TO IDENTIFY GAPS IN A
HEALTH DISPARITIES RESEARCH
PORTFOLIO.
TO ALSO DEVELOP HEALTH EQUITY
RESEARCH PRIORITIES FOR FIVE TO
TEN YEARS.
THE NATIONAL ADVISORY COUNCIL
WORKING GROUP ON HEALTH EQUITY
WAS CONVENED AND COMPRISED OF
OVER 30 RENOWNED HEALTH EXPERTS,
BOTH IN THE FIELD OF
NEUROLOGICAL DISEASE AND HEALTH
DISPARITIES.
AND THERE -- THEY WERE TASKED
WITH REGULATIONS.
THEY REVIEWED THE REPORT, ALSO
USED THE INPUT FROM TRANS-NIH
WORKING GROUP, PORTFOLIO
ANALYSIS AND REQUEST FOR
INFORMATION.
THEY ALSO CONDUCTED IN DEPTH
LITERATURE REVIEWS.
THE CAPSTONE OF THE PROCESS WAS
HEADWAY WORKSHOP, ALL COUNCIL
WORKING FOR FOR HEALTH EQUITY
PRESENTED SUMMARY TO LEADERSHIP
AND ALSO TO THE PUBLIC.
CLINICIANS RESEARCHERS, ADVOCACY
ORGANIZATIONS, COMMUNITY
MEMBERS, PATIENTS AND STAFF WERE
ALL ABLE TO COMMENT AND TO
PROVIDE ADDITIONAL FEEDBACK
DURING THIS WORKSHOP.
THE STRATEGIC PLANNING OUTPUTS
ARE DESCRIBED IN DETAIL WITHIN
10 MANUSCRIPT ACCEPTED BY THE
JOURNAL OF NEUROLOGY AND
EXPECTED TO BE PUBLISHED WITHIN
THE NEXT FEW WEEKS.
WE'RE REALLY EXCITED ABOUT THAT.
THE SUMMARIES WERE SUBMITTED,
CAPTURED WITHIN 18 OVERARCHING
RECOMMENDATIONS, SOME OF THESE
THEMES ARE DEPICTED IN THE WORD
CLOUD.
LARGELY THE THEMES COVERED A
VERY BROAD RANGE OF TOPICS
INCLUDING COMMUNITY ENGAGEMENT,
TRAINING AND EDUCATION,
COMMUNICATION, HEALTH EQUITY
RESEARCH.
AT THIS STAGE, THE NINDS IS NOW
FOCUSING ON THE STRATEGIC
IMPLEMENTATION OF THESE
RECOMMENDATIONS.
IN DIRECT RESPONSE TO THESE
RECOMMENDATIONS ONE OF THOSE
MAJOR EFFORTS IS NEW NINDS
COMMUNITY ENGAGED HEALTH EQUITY
RESEARCH IN NEUROSCIENCE
INITIATIVE WHICH IS A
MULTI-PRONGED APPROACH TO
ADVANCING COMMUNITY ENGAGED
HEALTH EQUITY RESEARCH ACROSS
ALL NEUROLOGICAL DISEASE AREAS
WITHIN POPULATIONS THAT
EXPERIENCE HEALTH DISPARITIES
AND TO SUPPORT COLLABORATION,
EDUCATION, AND CAPACITY BUILDING
TO CONDUCT SUCH RESEARCH.
THE INTERVENTION SDOH
COMMUNICATION AND FRAMEWORK
SUBCOMMITTEES ALL EMPHASIZE THE
IMPORTANCE OF ADVANCING
COMMUNITY ENGAGED RESEARCH IN
NEUROSCIENCE AND MEDICINE TO
FOSTER SUSTAINABLE HEALTH
DISPARITIES INTERVENTION AND TO
ALSO ACHIEVE HEALTH EQUITIES.
THESE RECOMMENDATIONS BROADLY
INCLUDE PRIORITIZING,
INCENTIVIZING RESEARCH INCLUSION
OF PARTICIPANTS AND RESEARCH
TEAMS WITH LIVED EXPERIENCE WITH
HEALTH DISPARITIES, IN
NEUROLOGICAL DISEASES.
AND MORE IMPORTANTLY, INVOLVING
COMMUNITIES AND THOSE
POPULATIONS THAT EXPERIENCE
HEALTH DISPARITIES IN THE
RESEARCH EARLY AND CONTINUOUSLY.
AS WELL AS DEVELOPING CURRICULA
THAT ADDRESS CHALLENGES IN
COMMUNITY ENGAGED RESEARCH.
FINALLY I'M ENTHUSED TO SHARE
ANOTHER MAJOR OUTPUT FROM THIS
PROCESS, WHICH WAS DEVELOPMENT
OF THE NINDS SOCIAL DETERMINANTS
OF HEALTH FRAMEWORK FOR
ADDRESSING HEALTH INEQUITIES.
WHICH HIGHLIGHT MODIFIABLE
SOCIAL DETERMINANTS OF HEALTH
RESEARCH, TARGET PATHWAYS THAT
SDOH AND HEALTH DISPARITIES
PROVIDE HEALTH INEQUITIES,
BROADLY ADAPTED IN NEUROSCIENCE
AND ALSO SERVE AS GUIDE TO
IMPROVE QUALITY OF NEUROLOGICAL
RESEARCH.
I THANK YOU FOR ALLOWING ME TO
PRESENT TODAY.
PLEASE USE THE FOLLOWING QR
CODES TO SUBSCRIBE TO OUR
LISTSERV AND VISIT OUR WEBSITE
FOR FUNDING OPPORTUNITIES, OUR
INITIATIVES, ANY ANNOUNCEMENTS.
NOW I WILL PASS THINGS OVER TO
MY COLLEAGUE DR. CHERYSE SANKAR.
THANK YOU.
>>THANK YOU FOR THE EXCELLENT
OVERVIEW.
I WILL BE SERVING AS MODERATOR,
I'M A PROGRAM DIRECTOR IN OFFICE
OF GLOBAL HEALTH AND HEALTH
DISPARITIES, WE'RE EXCITED TO
BRING TOGETHER SOME PARTNERS
FROM INDUSTRY AND NON-PROFIT TO
COLLECTIVELY DISCUSS THESE
CENTRAL ISSUES AROUND STRATEGIES
TO BOOST ENROLLMENT IN CLINICAL
TRIALS, PARTICULARLY THROUGH
THIS LENS OF COMMUNITY
ENGAGEMENT.
WE KNOW AND WE'VE HEARD THAT
THESE ARE OVERARCHING
CHALLENGES, WE DON'T INTEND OR
THINK WE CAN GET THROUGH ALL OF
THESE ARE IN THE 45-MINUTE
SESSION WE HAVE.
WE WANT TO IMTAMPA SIZE --
EMPHASIZE THIS IS ONE PART OF
THE DISCUSSION.
WE WANT TO HEAR FROM YOU AND MAY
NOT GET TO EVERYONE'S QUESTIONS
AND MAY NOT HAVE AS MUCH TIME
TOWARD THE END BUT IF YOU WANT
TO PROVIDE YOUR INPUT AND ANSWER
QUESTIONS WE'RE GOING TO BE
COLLECTING THOSE AT THE END, OUR
DIRECTOR DR. BENSON WILL COLLECT
THOSE AND LOOKING OVER THEM AND
CLOSING OUT OUR SESSION.
I WOULD LIKE TO INVITE OUR
SPEAKERS TO COME UP.
I'LL INTRODUCE THEM.
STEPHANIE MONROE, UsAgainst
ALZHEIMER'S, NATIONAL ADVOCACY
ORGANIZATION THAT'S BASED IN
WASHINGTON, D.C.
SHE DIRECTS AFRICAN AMERICANS
AGAINST ALZHEIMER'S FOUNDED IN
2013, THAT'S THE FIRST NATIONAL
NETWORK CREATED TO RESPOND TO
ALZHEIMER'S DISEASE DISPARATE
IMPACT ON AFRICAN AMERICANS,
RECENTLY PUBMED FANNED A PUBME-
PUBLISHED THE LACK OF
GENERALIZABILITY OF ALZHEIMER'S
RESEARCH MINORITIZED
POPULATIONS, CHECK THAT OUT.
WE INVITE YOU TO SIT AT THE
TABLE.
DR. PIERRE THEODORE, GLOBAL
PHARMACEUTICAL FIRM, ROCHE
GENENTECH, DISTINGUISHED CAREER
IN HEALTH CARE SERVICE DELIVERY,
INNOVATION, GLOBAL HEALTH IN
ACADEMIC AND PRIVATE SECTOR
ROLES.
WELCOME, DR. THEODORE.
THANK YOU.
MR. TONY PEARSON, SENIOR
DIRECTOR OF DIVERSITY AND
INCLUSION IN CLINICAL TRIALS AT
ELI LILLY, LEADS CLINICAL TRIAL
DIVERSITY OPERATIONS ACROSS ALL
AREAS AND MANAGES TEAMS
RESPONSIBLE FOR SITE START-UP
AND ENROLLMENT READINESS,
SUPPORTED THE DIABETES CORPORATE
AFFAIRS TEAM.
WELCOME TO THE PANELISTS.
I'LL HAVE A SEAT AS WELL BECAUSE
I DID NOT BRING SHOES TO STAND
HERE.
WE'LL START WITH THAT
INTRODUCTORY QUESTION.
I'LL TRANSITION HERE.
YOU HAVE TO PUSH THE BUTTON TO
START AND I DON'T THINK WE'LL
HAVE ANY ISSUES, THANKS TO THE
I.T. TEAM AS WELL.
WE'RE GOING TO KICK OFF WITH
SOME INTRODUCTORY QUESTIONS.
WE'D LIKE TO KNOW WITHIN YOUR
COMPANY AND ORGANIZATION, WHY IS
ADVANCING HEALTH EQUITY SO
IMPORTANT AND HOW DOES IT RELATE
TO DIVERSITY AND INCLUSION IN
CLINICAL STUDIES.
WE'LL START WITH MR. PEARSON.
>>FOR US AT ELI LILLY, I'LL
START WITH A COUPLE MOMENTS THAT
INSPIRE CHANGE.
SO, TWO THAT I RAISE WOULD BE
FDA'S DRAFT GUIDANCE IN 2020,
THEIR WORK STARTED WELL BEFORE
THEN BUT AS A FORMER
TRANSACTIONAL HEALTH LAWYER
THERE'S A CERTAIN LEVEL OF
MOTIVATION IN THE REGULATORY
ENVIRONMENT, SO APPLAUSE AND
APPRECIATION FOR THEIR
LEADERSHIP IN THAT SPACE.
WE EVOLVED FROM HEALTH EQUITY
PERSPECTIVE POST THAT TO HAVE
OUR FIRST TWO POSITIONS
DEDICATED TO DIVERSITY, EQUITY,
AND INCLUSION, I'M THE BETA
VERSION, THE NEXT EDITION OF
THAT EFFORT.
WE REALIZED THE SIZE OF OUR
ORGANIZATION WE NEEDED MORE OF A
FUNCTION THAN INDIVIDUAL
CONTRIBUTORS, SO WHAT I HAVE ARE
TEAM MEMBERS EMBEDDED WITHIN
EACH OF OUR THERAPEUTIC AREAS
DESIGNED TO DRIVE CLINICAL
TRIALS.
THE OTHER PIECE, WITH RESPECT TO
DEATH OF MR. FLOYD.
THE MURDER OF MR. FLOYD, ELI
LILLY & COMPANY SPARKED
EVOLUTION OF A RACIAL JUSTICE
CORPORATE INITIATIVE, FIVE
PILLARS, CENTRAL IS FOCUSED ON
HEALTH EQUITY.
MY TEAM AND SEVERAL MEMBERS,
EVEN WITH PRICING REIMBURSEMENTS
OR IS HEALTH EQUITY AND WITHIN
OUR CORPORATE AFFAIRS
DEPARTMENT, SPECIFICALLY SOCIAL
IMPACT.
ENROLLMENT IN DIVERSE
PERSPECTIVES IN THE TRIAL,
TRYING TO MAKE SURE WE HAVE
DIVERSITY AND INCLUSION IN
TRIALS TO GET MEDICATIONS TO
FOLKS WHEN THEY ACCESS THEM THEY
TRULY ARE SAFE AND EFFICACIOUS
FOR RESPECTIVE COMMUNITIES.
WE CAN'T SAY WE CARE ABOUT
EQUITY AND THE PART WE PLAY AND
STILL HAVE SUCH A WAYS TO GO TO
MAKE SURE WE'RE DELIVERING BY
MAKING SURE TRIALS ARE INCLUSIVE
AS THEY POSSIBLY CAN BE.
THE AREAS OF FOCUS ARE ACROSS
DIMENSIONS THAT WERE
ARTICULATED.
RACE AND ETHNICITY BUT ALSO
GENDER IDENTIFICATION,
ORIENTATION, PEOPLE WITH
DISABILITY MAKING SURE WE HAVE
ACCESS AND TACTICS TO SUPPORT
EACH ONE OF OUR THERAPEUTIC
AREAS TO MAKE SURE EACH
POPULATION AREAS THAT ACCESS TO
TRIALS AND ARE RETAINED.
>>THANK YOU VERY MUCH.
DR. THEODORE?
>>THANKS.
I'M DELIGHTED TO BE HERE.
I WANT TO COMMENT ON BEHALF OF
THE 110,000 EMPLOYEES AT ROCHE
AND GENENTECH WE'RE DELIGHTED TO
BE HERE AND LEARN ABOUT THE
PARTNERSHIP.
FROM OUR PERSPECTIVE HEALTH
EQUITY WHAT WE LABEL AS A CORE
OPERATING PRINCIPLE.
WE SAY THAT FOR A COUPLE
REASONS.
ONE WHICH IS SPECIFICALLY A
BUSINESS PRIORITY, WHICH IS TO
SAY THAT CLINICAL TRIALS IS
ESSENTIALLY THE LIFE BLOOD OF
OUR ORGANIZATION.
IT'S IMPORTANT WE BOTH INCLUDE A
WIDE RANGE OF PARTICIPANTS IN
THOSE CLINICAL TRIALS, BUT
MOREOVER THAT WE ENSURE
OURSELVES THE DRUGS WE BRING TO
MARKET ARE SAFE AND EFFECTIVE IN
ALL POPULATIONS, CLINICAL TRIAL
DIVERSITY IS AN ESSENTIAL PART
OF OUR DRUG DEVELOPMENT
STRATEGY.
THE SECONDHAND, IT'S QUITE
SIMPLY A MORAL INTERACTIVE,
PLAYING A CRITICAL ROLE IN THE
HEALTH CARE SYSTEMS GLOBALLY,
NOT TO TAKE QUITE SERIOUSLY THE
IDEA OF BEING INCLUSIVE AND
REPRESENTATIVE IN OUR CLINICAL
TRIALS AND DRUG DEVELOPMENT AND
MARKETING AND COMMERCIALIZATION
STRATEGIES WOULD BE A MORAL
FAILURE ON OUR PART.
THERE ARE WE'VE CONTINUED TO
BUILD OUT EFFECTIVELY PATIENT
INCLUSION AND HEALTH IMPACT
FUNCTIONALITIES TO LIVE INTO THE
MORAL MISSION FOR GENENTECH AND
ROCHE, BEING OUR PARENT COMPANY.
>>AND MS. MONROE?
>>I THINK IT'S A GREAT QUESTION
BECAUSE AS A PATIENT ADVOCACY
ORGANIZATION WHEN WE FOUNDED 13
YEARS AGO ONE OF THE FIRST THING
DID I IS LOOKED AT DATA.
I'M A DATA WONKY PERSON.
I WANTED TO UNDERSTAND WHAT WAS
GOING ON IN THE AFRICAN AMERICAN
LATINO POPULATION WITH
ALZHEIMER'S.
AND IT WAS VERY QUICKLY
UNDERSTOOD BY ME AND BY OTHERS
THAT WE WERE IN FACT LIKE SO
MANY OTHER DISEASES TWICE AS
LIKELY TO DEVELOP ALZHEIMER'S AS
OTHERS.
ALSO QUICKLY LEARNED THAT WE
WERE WAY UNDERDIAGNOSED, ABOUT
40% OF PEOPLE LIVING, WALKING,
BREATHING, HAVING CAREGIVER
BURDEN AND EVERY EXPERIENCE WERE
NOT AWARE THEY HAD THE DISEASE,
DIDN'T KNOW WHAT TO DO, WE
DIDN'T HAVE GOOD PATHWAYS,
DIDN'T HAVE AWARENESS, THERE WAS
STIGMA AND SHAME.
THE ONLY WAY TO ADDRESS THAT WAS
GET OUT IN THE COMMUNITY.
AN AGENCY LOOKING TO IDENTIFY
BARRIERS AND REACH PEOPLE WE
FORMED THE AFRICAN AMERICAN
NETWORK AGAINST ALZHEIMER'S, AND
LATINO NETWORK, TO BE A VOICE TO
THOSE POPULATIONS.
WE BEGAN TO LOOK AT STRATEGIES
REACHING POPULATIONS INITIALLY
WITH JUST AWARENESS BECAUSE BACK
IN 2010 PEOPLE REALLY WEREN'T --
THEY WERE DOING ALZHEIMER'S AS
THE C WORD, THE CANCER WORD,
DIDN'T WANT TO TALK ABOUT IT.
EVEN TODAY MEAN PEOPLE STILL
BELIEVE IT'S A NORMAL PART OF
AGING.
THAT BECAME A CORE STRATEGY TO
GET OUT IN THE COMMUNITIES,
WE'LL TALK ABOUT WHAT WE DID AND
BEGIN TO RAISE THIS ISSUE IN
COMMUNITIES.
LOOKING AT DATA AND HOW
IMPORTANT IT IS FOR NOT JUST
PEOPLE BEING ABLE TO ACCESS
TREATMENT AND CERTAINLY THE NEW
TREATMENTS COMING, BUT TO MAKE
SURE BIOMARKERS INCLUDE PEOPLE
SO AS WE'RE GOING OUT AND
DEVELOPING BLOOD TESTS,
DIFFERENT WAYS OF IDENTIFYING
DISEASES, INCLUDED A LARGE
ENOUGH POPULATION, DATA IS
RELEVANT.
SAME THING WITH OTHER TESTS LIKE
A LOT OF TESTS WE DO ARE
UNFORTUNATELY BIASED AGAINST
PEOPLE WHO HAVE A CERTAIN
READING LEVEL OR HEALTH LITERACY
LEVEL.
WE WORK WITH THEM TO USE THE
RIGHT KINDS OF WORDS, TO MAKE
SURE PEOPLE HAVE AN OPPORTUNITY
TO GET THE EQUITY, TO ACCESS THE
KNOWLEDGE, GET THE RIGHT
DIAGNOSTICS, AT THE RIGHT TIME,
IN THE COMMUNITY, WITH THE RIGHT
VOICES.
THAT BECAME THE CORE OF OUR
ORGANIZATION, AN EQUITY FOCUS.
WE INCLUDED WOMEN IN OURS.
AND IT WAS REALLY IMPORTANT
BECAUSE GENETICALLY WE'RE SEEING
DIFFERENCES IN WOMEN AS WE ARE
THE GENETICS OF AFRICAN
AMERICANS AND LATINOS AS WELL AS
OTHERS.
>>THANK YOU.
THAT WAS A WONDERFUL INTRO TO
THINKING ABOUT THE IMPORTANT
PRINCIPLES FOR WHY WE FOCUS ON
ADVANCING HEALTH EQUITIES, SOME
OF THE COMMUNITY ENGAGEMENT
PRINCIPLES WE WANT TO
INCORPORATE ABOUT INCLUDING
BIDIRECTIONAL COMMUNICATION AND
UNDERSTANDING THE EDUCATION OF
THE COMMUNITY AND THE BELIEF AND
CULTURE BEHIND THAT.
SO I'D LIKE US TO THINK NOW AND
TALK ABOUT SOME REAL WORLD
EXAMPLES THAT YOU'VE UTILIZED IN
YOUR COMPANIES AND ORGANIZATIONS
TO REALLY APPLY THE KNOWLEDGE
THAT WE HAVE IN UNDERSTANDING
HOW TO DIVERSIFY SO IF YOU COULD
PROVIDE A CASE EXAMPLE IN YOUR
PARTICULAR EXAMPLE THEN THAT
WOULD BE GREAT.
WE'LL START WITH MS. MONROE.
LET'S FOLLOW UP ON THE
CONVERSATION YOU WERE HAVING
EARLY ABOUT ENGAGING THE
COMMUNITY.
>>SO, I SHOULD SAY I'M A LAWYER
BY TRAINING.
I'M A PUBLIC POLICY PERSON, 25
YEARS ON CAPITOL HILL.
AND HAVE NO PARTICULAR IDEAS
ABOUT HOW TO REACH COMMUNITIES
EXCEPT WHAT I KNEW WORKED FOR ME
AND IN MY FAMILY.
I GREW UP IN BALTIMORE.
WORKED IN D.C.
I LIVE IN VIRGINIA.
MY BROTHER'S A DOCTOR.
WE OFTEN JUST BANTER ABOUT THESE
CONVERSATIONS.
AND IT SEEMED TO ME THAT, YOU
KNOW, IF SOMEONE INVITED ME TO A
LECTURE ABOUT A DISEASE AND
RAISING AWARENESS I WOULD BE
LIKE, NO.
IF SOMEONE INVITED ME TO COME
MAYBE TO AN ICE CREAM SOCIAL, AS
I'M EATING MY CHOCOLATE DIP ICE
CREAM, AND THEY WANT TO TALK TO
ME ABOUT HOW BAD SUGAR IS, I
WOULD STILL SAY NO.
BUT IF THEY INVITED ME TO A
PLAY, SOMETHING WHERE I COULD
BRING MY FRIENDS, MAYBE IT WAS
FREE, FELT LIKE A RED CARPET
EVENT, MUSIC, MAYBE GOSPEL,
LAUGHTER, OKAY, LET'S DO THAT.
SO WORKED WITH A PLAYWRIGHT,
DEVELOPED A PLAY CALLED "FORGET
ME NOT," TAKING IT TO 27 CITIES,
REACHED 40,000 PEOPLE.
SURVEYED PEOPLE THERE, TO FIND
OUT ABOUT THEIR EXPERIENCE.
WHAT THEY KNEW.
ONE OF THE FIRST QUESTIONS WE
ASKED HAS YOUR DOCTOR EVER
TALKED TO YOU ABOUT BRAIN
HEALTH?
NO.
OVERWHELMING, NO.
NO ONE TALKED TO ME ABOUT IT.
WOULD YOU BE INTERESTED IN
PARTICIPATING IN CLINICAL
RESEARCH THAT COULD HELP YOU,
YOUR FAMILY MEMBERS, HELP
SOCIETY LEARN MORE ABOUT THIS
DISEASE AND WHAT THE LIKED LIKE?
80% SAID YEAH, WE'D BE
INTERESTED IN LEARNING ABOUT
THAT.
I ASKED HAS ANYONE EVER ASKED
YOU TO PARTICIPATE IN CLINICAL
RESEARCH?
EVERYBODY TALKS ABOUT HOW BLACKS
WON'T PARTICIPATE, LATINOS WON'T
PARTICIPATE.
80% OF PEOPLE SAID THEY HAD
NEVER BEEN ASKED.
WE CAN DEAL WITH THAT, RIGHT?
WE TO GET INDUSTRY AND
PHYSICIANS COMFORTABLE WITH
RAISING THAT ISSUE.
FEEL LIKE THEY ARE PARTNERS.
WE EMPLOYED THAT STRATEGY USING
OUR PLAY, BROUGHT INFORMATION TO
THE COMMUNITY, PARTNERED WITH
THE COMMUNITIES, ASKED THEM WHAT
THEY WOULD LIKE TO SEE AS PART
OF THIS, BROUGHT IN LOCAL
SERVICES, BECAUSE WE'RE NOT A
LOCAL ORGANIZATION.
WE PROVIDED A PLATFORM, PROVIDED
ENTERTAINMENT, PROVIDED FOOD,
GATHERED PARTNERS TOGETHER SO IT
BECAME THEIR EVENT.
THEY DON'T KNOW ME, I'M
WASHINGTON, D.C.
IF I BRING THEM THIS STUFF,
OKAY, RIGHT.
WE LEFT BEHIND PARTNERS TO WORK
TOGETHER.
IT WAS VERY EFFECTIVE.
DOING SOMETHING YOU WOULD LIKE
TO DO, AND RECEIVING INFORMATION
LIKE YOU WOULD LIKE TO RECEIVE
IT, INCLUDE PEOPLE WHO LOOK LIKE
THE PEOPLE YOU'RE TRYING TO
REACH, MAKING IT REALLY FUN AND
INFORMATIONAL, AND NOT JUST SAY
HERE'S A PRESCRIPTION, SEE 'YA
LATER, THAT WILL BE VERY
EFFECTIVE.
>>THANK YOU, FANTASTIC EXAMPLE
OF ENGAGING THE COMMUNITY,
WORKING WITH THE LOCAL
PARTNERSHIPS.
I HEARD A LOT OF THIS AS WE WERE
GOING THROUGH THIS IN THE
NON-PROFIT SESSION.
REALLY IN LINE WITH WHAT WE'RE
HEARING.
DR. THEODORE?
>>SURE.
I MIGHT PUT CONTEXT BY SAYING
FROM OUR PERSPECTIVE IF WE
CONTINUE TO DO THE SAME THINGS
WE'VE DONE FOR A CENTURY OR SO
WE'LL END UP WITH ROUGHLY THE
SAME RESULTS.
AND SO FUNDAMENTALLY WE'VE TRIED
TO TAKE NEW STRATEGIES FOR
REACHING PATIENTS, MAKING SURE
WE'RE BEING INCLUSIVE IN
CLINICAL RESEARCH, BUT IN ORDER
TO TAKE A NEW STRATEGY WE NEED
TO HEAR FROM WELL-INFORMED
VOICES.
SO WE NEED TO CREATE
CONSORTIUMS, CREATE ALIGNMENTS
AMONG NON-PROFITS, PATIENT
ADVOCACY GROUPS, AND GO OUT TO
CLINICAL TRIALS CENTERS, FOR
EXAMPLE, WITH A PROVEN HISTORY
IN MEETING NEEDS OF
TRADITIONALLY UNDERSERVED
POPULATIONS.
SO, ONE OF THE EFFORTS THAT WE
LAUNCHED NOW GOING ON THREE
YEARS AGO WAS A SITE ALLIANCE,
CLINICAL TRIAL SITES
SPECIFICALLY WITH A TRACK RECORD
OF REACHING PRIMARILY HISPANIC,
AFRICAN AMERICAN, RURAL POOR
PATIENTS, SO WE ESSENTIALLY
WOULD BE GETTING NOT ONLY CLOSER
TO THE PATIENTS WE WANTED TO
ADDRESS BUT BY INTERACTING
DIRECTLY WITH THE PRINCIPAL
INVESTIGATORS WOULD START TO GET
INSIGHTS ABOUT WHAT WERE WE
MISSING IN ADDRESSING NEEDS OF
PATIENTS TO BE MORE INCLUSIVE
WITH RESPECT TO OUR CLINICAL
TRIAL EFFORTS.
I WOULD SAY ONE OF THE FIRST
STRATEGIES BROADLY SPEAKING WAS
TO BE MORE OPEN TO LISTEN MORE
DIRECTLY, REALLY FROM PEOPLE WHO
HAD BOOTS ON THE GROUND AND
ADDRESSING NEEDS OF
TRADITIONALLY UNDERSERVED
POPULATIONS.
>>AT LILLY WE PROVIDE OURSELVES
ON BEING BOLD, FAST, CLEAR.
DURING COVID, HEIGHT OF COVID,
WE WERE COMMITTED TO MAKING SURE
THAT WE PROVIDED ACCESS TO
CLINICAL TRIALS FOR VULNERABLE
POPULATIONS, THE FISSURES WE
KNOW TO BE CHASMS, FOCUS ON
TAKING CARE TO PEOPLE LIVING IN
NURSING HOMES, TO ENSURE WE WERE
ABLE TO CONDUCT CLINICAL TRIALS
THERE.
THE IDEA WAS TO OUTFIT A
RECREATION VEHICLE AND DRIVE TO
NURSING HOMES.
ONE WITH A DIVERSE POPULATION,
THE AMOUNT OF TRUST BEING
INVITED CREATED AN OPPORTUNITY
WE WANTED TO CONTINUE.
WE'VE EVOLVED OUR MOBILE
RESEARCH UNIT, NOW VERSION 3, TO
CONDUCT PRE-SCREENING,
SCREENING, LABS, DRIVE ACROSS
THE COUNTRY, ALSO ABILITY
FOCUSED ON PATIENTS LIVING WITH
DISABILITIES TO SORT OF TAKE
THINGS INSIDE THE MOBILE
RESEARCH UNIT AND PUT THEM
OUTSIDE SO PEOPLE HAVE NO ISSUE
ACCESSING THE UNIT.
WE'RE BEING INVITED TO LEVERAGE
UNITS FOR PRE-SCREENING ACROSS
MULTIPLE CLINICAL TRIALS.
SO WE CONTINUE TO TRY TO DO
THOSE THINGS, GET IN THOSE
SPACES.
NOT EVERY OPPORTUNITY IS GOING
TO BE SUCCESSFUL.
PART OF THE JOURNEY IS TO FAIL
FAST AND BE IN THE RIGHT SPACES.
AS A SMALL EXAMPLE, LAST WEEK WE
HAD THE INDIANA BLACK AND
MINORITY HEALTH FAIR, IN THE
CITY OF INDIANAPOLIS, STATE OF
INDIANA, WE'RE WELL KNOWN.
WE'RE GOOD CORPORATE SPONSORS,
DOING THE THINGS WE'RE SUPPOSED
TO DO.
UNTIL LAST YEAR WOULD SHOW UP 84
VENDORS, WE WOULD SHOW UP AND
PASS OUT BAGS.
WE'RE AT A HEALTH FAIR.
WHY ARE WE NOT LEVERAGING
INNOVATION TO DO SOME SCREENING?
I APPROACHED LEGAL COLLEAGUES, I
PICK TWO FIGHTS A WEEK WITH
PEOPLE I SHOULD SAY.
ONLY BECAUSE I'M TRYING TO
CHALLENGE US TO DO THINGS
DIFFERENT, NOT THAT WE'RE AT
ODDS.
THEY HAVE A WAY OF OPERATING.
WE'RE COGNIZANT OF REGULATORY
ENVIRONMENT AND WE NEED TO MOVE
THE SPEED.
CAN WE LEVERAGE OUR MOBILE
RESEARCH UNIT TO DO
PRE-SCREENING NEXT TO THE
GIVEAWAYS?
NO, THAT LOOKS LIKE PATIENT
INCENTIVIZATION.
I WENT TO CORPORATE TEAM, WE'RE
GOING TO GET RID OF THIS TO
TRADITIONAL VOLUNTEERS, WE NEED
TO SHOW UP AND DO PRE-SCREENING,
CLINICAL TRIAL GENERAL EDUCATION
AND SCREENING AND HELP THE
COMMUNITY.
WE DID A PANEL THAT FROM MY
ESTIMATION FAILED.
THERE WAS TRAFFIC, MOST PANELS
FAILED.
THE FAILURE THAT PRECEDE US WAS
HEALTHY COOKING ON JAMBALAYA,
WELL ATTENDED.
THEY LEFT, WE DIDN'T HAVE THE
SAME DRAW.
BUT WE WERE IN THE SPACE.
I GO BACK, HEY, THIS IS HOW IT
WENT, WE START PLANNING FOR NEXT
YEAR.
HOW DO WE PARTNER DIFFERENTLY
WITH THE INDIANA STATE
DEPARTMENT OF HEALTH TO MAKE
SURE YEARS FROM NOW EVENTS ARE
WELL OILED, WELL TAILORED.
THAT'S HOW WE SHOULD BE
DEPLOYING VERSUS SHOWING UP AND
SAYING HI, WE'RE LILLY.
THANK YOU.
EXCELLENT EXAMPLE AS WELL.
THIS REMINDS ME OF THE COMMENT
MADE FROM THE PREVIOUS PANELISTS
ABOUT THE GIVEBACK, RIGHT?
AND THAT IS A TANGIBLE BENEFIT
BRINGING THE PRE-SCREENING TO
THAT HEALTH FAIR INSTEAD OF
PASSING OUT INFORMATION.
HOW ARE YOU CREATING
BIDIRECTIONAL COMMUNICATION AND
PROVIDING A BENEFIT TO THE
COMMUNITY THAT YOU WISH TO SERVE
AND BRING INTO THESE STUDIES?
NOW WE'VE TOUCHED ON SOME OF THE
BIG CHALLENGES AND BARRIERS.
I HEARD TRUST AS AN ISSUE.
WE KNOW THAT'S ABSOLUTELY AN
ISSUE.
I'VE HEARD ACCESS AS AN ISSUE.
MAYBE IN THE NEXT SEGMENT WE CAN
JUST TALK ABOUT WHAT YOU SEE AS
KEY BARRIERS AND CHALLENGES IN
TRYING TO DO YOUR JOBS, TRYING
TO INCREASE DIVERSITY AND
INCLUSION AND ENGAGE THOSE
COMMUNITIES.
AGAIN, YOU GAVE SOME EXAMPLES.
IF YOU HAVE SOME MORE, LOVE TO
HEAR THOSE.
MAYBE WE'LL START WITH DR.
THEODORE AND GO DOWN.
>>I'LL KEEP IT SIMPLE.
I THINK IN MANY HEALTH CARE
SYSTEMS THERE ARE SIGNIFICANT
BIASES AGAINST POPULATIONS OF
PATIENTS, OFTEN MANIFEST IN NOT
BEING ASKED TO ENGAGE IN
CLINICAL TRIALS, PRESUMPTIONS
MADE ABOUT VARIOUS GROUPS OF
PATIENTS, AS A CONSEQUENCE
RELATIVE REPRESENTATION IN
CLINICAL TRIALS IS OFTEN BELOW
WHAT WE ANTICIPATE.
ONE FUNDAMENTAL LEVEL THERE ARE
SIGNIFICANT BIASES INSIDE THE
HEALTH CARE SYSTEM WE'RE TRYING
TO ADDRESS THROUGH HEALTH CARE
PROVIDER EDUCATION, THROUGH
VARIOUS FORMS OF BIAS TRAINING,
WE'VE ENGAGED IN WITH OUR
CLINICAL TRIALS SPECIALISTS.
BUT BEYOND THAT, I THINK THERE
ARE OFTEN SIGNIFICANT FINANCIAL
TOXICITIES THAT GO ALONG WITH
CLINICAL TRIALS.
WE TAKE IT FOR GRANTED THAT
PATIENTS WHEN ASKED ABOUT HAVE
THE CAPACITY TO PARTICIPATE.
FOR EXAMPLE, THEY MAY BE ASKED
TO COME IN EXTRA TIME FOR CT
SCANS AND MRIs, AS SIMPLE AS
ASKING THEM TO PAY FOR PARKING
OR TRAVEL.
THERE ARE BARRIERS HOW MUCH WE
CAN FINANCIALLY SUPPORT THE
PATIENTS, THAT REPRESENTATIVES A
SIGNIFICANT BARRIER TO MANY
OFTEN UNDERSERVED MINORITIZED
MARGINALIZED POPULATIONS.
WHEN WE PUT SORT OF THE INHERENT
BIASES IN AND ADD TO FINANCIAL
TOXICITY, CONSPIRING, FORMS A
SIGNIFICANT BARRIER FOR
ENGAGEMENT.
>>WE'RE FOCUSED IN THREE AREAS,
INCREASING UNDERACCESS,
PARTNERING WITH INVESTIGATORS,
DIVERSE AND ELEVATING THOSE
PARTICIPATING IN THE STUDY
SPECIFICALLY.
WE KNOW JUST UNDER 6% OF
PHYSICIANS, BLACK PHYSICIANS,
EVEN FEWER CLINICAL TRIALISTS,
BUT THEY PLAY OUTSIZE ROLE IN
TRUST.
WE WANT TO GIVE PATIENTS ACCESS
OR SUPPORTING THEIR ACCESS TO BE
ABLE TO BE RECRUITED AND
RETAINED IN STUDIES.
KNOWING WE HAVE TO WORK WITH
PHYSICIANS THAT DON'T HAVE THE
SAME DEMOGRAPHIC MAKEUP AS
PATIENTS THEY ARE SERVING, WITH
RESPECT TO COMMUNITY, PART OF
THE COMMUNITY ENGAGEMENT IS
PARTNERING WITH THE NETWORK FOR
HEALTH -- EXCELLENCE IN HEALTH
INNOVATION OUT OF BOSTON, PARDON
ME.
CREATING A COMMUNITY FOCUSED
RESEARCH ORGANIZATION, ALIGNMENT
OF 30 STAKEHOLDERS FROM ACROSS
INDIANA, IN PART BECAUSE IF WE
CAN'T DO IT IN OUR OWN STATE HOW
WOULD YOU BELIEVE WE COULD DO IT
ELSEWHERE?
WE GET THOSE FOLKS, FAITH
COMMUNITY, ACADEMIA, QUALIFIED
HEALTH CENTERS, REPRESENTATIVES
OF YOUTH ORGANIZATIONS, THE
LIKE, YMCA, TO COME AND ARRIVE
AT BARRIERS THAT IF WE OVERCOME,
ONE, TOGETHER, THAT WOULD ENABLE
ACCESS ACROSS THE STATE.
URBAN COMMUNITY, RURAL
COMMUNITY, YOU PICK, WE HAVE
ABILITY TO ENABLE ACCESS.
SOME THINGS THAT SURFACED FROM
CONVERSATIONS ARE ABOUT MISTRUST
OF THE HEALTH CARE CONTINUUM OR
INTERACTION WITH THE SYSTEM.
WE'RE GOING TO SEND SOMEONE BACK
TO A SYSTEM THAT'S ROUTINELY
FAILED THEM TO CONDUCT A
CLINICAL TRIAL THAT'S HARD.
THE OTHER PIECE WE'RE GETTING IS
TRUSTED INTERMEDIARY.
I HAVE A RESPONSIBILITY, I CAN'T
BE IN THE COMMUNITY AS MUCH AS
THEY NEED TO SEE ME.
PEOPLE FROM THOSE COMMUNITIES,
ADVOCATES, NAVIGATORS CAN BE
EMPOWERED WITH INFORMATION THAT
CAN SUPPORT CONVEYING
INFORMATION, YESTERDAY WE TALKED
ABOUT ABOVE INFORMED CONSENT TO
INCLUSION EXCLUSION CRITERIA,
THAT EXPLANATION, HOW DO WE
EMPOWER AND ENGAGE PEOPLE TO
HAVE THEM FAMILIAR WITH CLINICAL
TRIALS IN THE PROCESS.
WE CONTINUE TO SURFACE THOSE
BARRIERS AND MAKE SURE WE'RE
ALIGNING ON WHAT THOSE ARE AND
EMPOWER THOSE PARTICULAR
COMMUNITY GROUPS THAT REACH
THEIR OWN COMMUNITY.
>>ALZHEIMER'S IS A SMALL
ORGANIZATION, WE PUNCH ABOVE OUR
WEIGHT.
I AGREE WITH WHAT THESE GUYS
HAVE SAID.
FROM A PATIENT ADVOCACY
PERSPECTIVE, BUT ONE UNIQUE
THING IS TOO REALLY USE DATA IN
A WAY FOR PATIENTS WITH
ALZHEIMER'S DISEASE.
WORKING IN PUBLIC POLICY,
POLITICIANS ARE USED TO SEEING
LOTS OF RAW NUMBERS BUT YOU HAVE
TO CHALLENGE THEM TO REMEMBER
THERE'S AN ACTUAL PERSON WHO HAS
A LIVED EXPERIENCE THAT GOES
BEYOND THE PIECE OF PAPER.
STORY-TELLING AND PROMOTING
PEOPLE TO COME OUT OF THE
SHADOWS WE'VE WORKED TO DO,
CATALOGED IN VIDEOS WE USE.
WE DEVELOPED A NATIONAL
ALZHEIMER'S DISEASE INDEX.
WE'VE USED -- ONE OF THE MOST
EXCITING THINGS THAT I'VE HAD
THE PLEASURE OF DEVELOPING,
USING MEDICARE DATA SINCE MOST
DIAGNOSED WITH ALZHEIMER'S ARE
ON MEDICARE, ABLE TO IDENTIFY
DOWN TO A ZIP CODE LEVEL WHERE
PEOPLE LIVE WHO HAVE BEEN
DIAGNOSED WITH ALZHEIMER'S
DISEASE, MAP AND CREATE
CONGRESSIONAL REPORT WHERE WE
CAN SHOW HERE'S YOUR DISTRICT,
HERE'S THE NUMBER OF PEOPLE WHO
ARE ON MEDICARE, HERE IS
HOSPITALIZATION COST, HERE ARE
NUMBER OF PEOPLE WITH
ALZHEIMER'S ON MEDICARE, THEIR
COST.
WE NEED THIS INVESTMENT IN YOUR
COMMUNITY.
OVERLAY THAT WITH DATA ON, FOR
EXAMPLE, TO GET A REALLY GOOD
DIAGNOSTIC FOR ALZHEIMER'S YOU
NEED A PET SCAN, THERE'S 2500 IN
THE COUNTRY.
WE HAVE THEM IN OUR INDEX.
WE CAN SHOW HERE IS WHAT
PATIENTS ARE BUT THERE AREN'T
PET SCANS NEARBY.
THERE AREN'T ENOUGH NEUROLOGISTS
NEARBY.
HERE IS WHERE THE PATIENTS ARE
BUT THERE AREN'T ALZHEIMER'S
DISEASE RESEARCH CENTERS.
WE USE IT TO HAVE PEOPLE DEVELOP
INFRASTRUCTURE.
IF A COMPANY, AND SOME USED OUR
SITE TO SAY WE NEED TO FIGURE
OUT WHERE WE SHOULD STAND UP THE
NEXT CLINICAL TRIAL IF
RECRUITING THESE POPULATIONS ARE
IMPORTANT.
AGAIN, WE CAN GO TO THE ZIP
CODE, STREET, WHERE YOU SHOULD
GO, TELL YOU WHAT THE HOSPITAL
SITUATION LOOKS LIKE.
WE CAN TELL YOU WHERE YOU'VE GOT
A BIG HOSPITAL, LOTS OF PEOPLE,
I'M SORRY, LOTS OF GREAT
INFRASTRUCTURE, SCANS, MRIs,
TONS OF DOCTORS, A LOT OF MONEY,
THAT'S NOT WHERE BLACKS AND
LATINOS GO.
THEY GO ACROSS STREET WHERE NONE
OF THAT EXISTS.
WE'RE THE PARTNER.
WE'VE BEEN ABLE TO, USING DATA,
FOR ACTION.
>>YOU MADE AN IMPORTANT POINT.
I THOUGHT I MIGHT BUILD ON IT.
YOU MENTIONED HOW OFTEN YOU HAVE
NON-PROFITS THAT THEN ARE ABLE
TO PRESENT TO FALL LEADERSHIP
AND CONGRESS, FOR EXAMPLE.
THAT'S A GREAT EXAMPLE OF WHERE
BIG PHARMA SOMETIMES AND SMALLER
NON-PROFITS CAN COME TOGETHER.
WE HAVE THESE PLATFORMS FOR
LOBBYING AND CONNECTING, TO
CONNECT TO NON-PROFITS ALLOWS
VOICES OF INDIVIDUAL PATIENTS TO
BE AMPLIFIED, SYMBIOSIS BETWEEN
BIG PHARMA AND THESE GROUPS.
>>THANK YOU FOR BRINGING THAT
UP.
I'M HEARING A LOT OF EXAMPLES OF
WHAT WE COLLECTIVELY TERM SOCIAL
DETERMINANTS OF HEALTH, THINKING
ABOUT WHERE WE WORK, LIVE, PLAY.
AND HOW THAT IMPACTS OUR HEALTH
AND OUR ACCESS AND OUR ABILITY
TO REALLY ADVANCE THE EQUITY
WITHIN THE COMMUNITIES THAT WE
LIVE IN.
STARTING TO THINK ABOUT FOR THE
NON--- THE FOLKS IN THE AUDIENCE
HERE, NON-PROFIT ORGANIZATIONS
THAT WE REALLY WANT TO THINK
ABOUT WHAT IS VALUABLE
INFORMATION THAT WE CAN THINK
ABOUT FROM THE PHARMA
PERSPECTIVE AND USE THAT
KNOWLEDGE TO REALLY HELP YOU ALL
AND COLLABORATE AND FORM
NETWORKS.
I KNOW THERE ARE A NUMBER OF
ORGANIZATIONS WITHIN THE
AUDIENCE THAT FOCUS, FOR
EXAMPLE, ON RARE DISEASES,
THAT'S A PARTICULAR CHALLENGE
WHEN IT COMES TO INCREASING
DIVERSITY AND INCLUSION AND
WHAT'S ALREADY CONSIDERED RARE
DISEASES, SO I WONDERED IF --
THIS IS OPEN TO ANY, IT'S OKAY
IF YOU DON'T HAVE RESPONSES, BUT
IF HAVE YOU INSIDER THOUGHTS
ABOUT HOW WE CAN INCREASE
DIVERSITY AND INCLUSION IN RARE
DISEASES AND GET ACCESS TO
COMMUNITIES WHEN IT REALLY MAY
BE A VERY SMALL COMMUNITY TO
START.
>>I'LL MAKE A GENERAL COMMENT,
WHICH IS GETTING ACCESS MEANS
MAKING SURE THAT YOUR MATERIALS
ARE ACCESSIBLE, RIGHT?
IT'S NOT JUST A MATTER OF HOW
MANY LEAFLETS DO WE GET TO THE
COMMUNITY.
IT'S DOES THE COMMUNITY READ
WHAT WE GIVE THEM?
IS IT WRITTEN AT A LEVEL SO THEY
CAN UNDERSTAND?
IS IT CULTURALLY APPROPRIATE?
SO LOOKING AT THOSE MATERIALS,
READING FROM BASICALLY A FOURTH
TO SIXTH GRADE READING LEVEL,
EVEN COLLEGE GRADUATES ARE
READING AT SIXTH GRADE READING
LEVEL, THAT'S THE TRUTH.
MAKING SURE IT HAS COLOR
PICTURES, IT'S MORE THAN JUST A
FRONT AND BACK PAGE, MAKE IT
SOMETHING THAT LOOKS NICE.
WE WERE LOOKING AT, FOR EXAMPLE,
AN ORGANIZATION THAT WAS
PROVIDING MATERIALS, IT WAS
INCREDIBLY SCIENTIFIC.
I'M READING IT, I DON'T KNOW
WHAT THIS MEANS.
WHY ARE YOU USING THIS ACRONYM?
YOU SAY YOU'RE TRYING TO REACH
THE AFRICAN AMERICAN POPULATION
BUT YOU'RE USING PEOPLE WHO ARE
CLEARLY ETHIOPIAN AS PICTURES.
IT'S JUST BEING SENSITIVE AND
AWARE OF WHAT THAT IS SO THAT,
AGAIN, IT WILL BE APPEALABLE TO
THOSE FOLKS THAT.
CAN HAPPEN ANYPLACE.
RARE DISEASES ARE UNIQUE OBVIOUS
LI BECAUSE YOU HAVE A GROUP OF
PEOPLE BUT RELY ON NETWORKS TO
INTRODUCE YOU TO OTHER PEOPLE
THAT YOU NEED TO CONTACT WITH
WOULD BE MY SUGGESTION.
THANK YOU..
>>MR. PEARSON?
>>TWO THINGS.
THERE CAN BE UNIQUE NEED AND
FOCUSES BETTER HERE BUT THINK
ABOUT THE COLLECTIVE NEED, A SET
OF COMMON ACCESS AND EDUCATION
AND INFRASTRUCTURE NEEDS THAT
COLLABORATION, IT'S BEEN
EXPRESSED COMING TOGETHER WITH
INDUSTRY AND SOLUTIONIZING
TOGETHER IS EXTREMELY VALUABLE.
THERE'S AN ELEMENT OF RESOURCE
THAT WE HAVE, DEXTERITY WE CAN
PROVIDE.
WHAT I'D SAY WHAT ARE THE
COMMON -- WHEN I THINK ABOUT --
YOU TALK ABOUT ALIGNING WITH
NON-PROFIT INDUSTRY FROM POLICY
PERSPECTIVE, THE CLINICAL TRIAL
COMPENSATION IS INCOME FOR FOLKS
ON PUBLIC PROGRAMS,
PARTICIPATING IN PUBLIC
PROGRAMS, I WOULD IMAGINE THAT'S
A SPACE THAT MOST OF US ARE
CONCERNED ABOUT.
TO WE HAVE ABILITY TO COME
TOGETHER AND SUPPORT?
THERE'S NO BETTER WAY TO BUILD
TRUST THAN TO PARTNER WITH AN
INSTITUTION THAT DOESN'T HAVE
ANYTHING IN ITS PIPELINE FOR
WHAT YOU'RE WORKING ON BUT WE'RE
STILL HERE TO HELP AND RAISE
THAT STANDARD.
WHAT ARE THE COMMON COMPONENTS
ACROSS THAT CAN BE SUPPORTED?
THE OTHER PIECE IS YOU KNOW MORE
ABOUT YOUR POPULATION THAN WE
COULD BEGIN TO.
SO COMING INTO THE ORGANIZATION,
SOMETHING ABOUT THAT.
I HOSTED A GRAND ROUNDS FOR ELI
LILLY, THE JOURNEY ACROSS THE
GLOBE.
HAVING EXTERNAL PERSPECTIVES
TAKE ACADEMIC AND TECHNICAL MIND
AND MAKES IT DIFFERENT, BECAUSE
WE CAN GET EVEN BETTER.
YOU ALL COMING AND GIVING
INSIGHT ABOUT WHAT WOULD HELP
YOU IN TERMS OF UNDERSTANDING
PATIENT JOURNEY, CAREGIVER
JOURNEY, THOSE THINGS ARE
VALUABLE TO US AUGUST.
SO WE HAVE ABILITY TO PARTNER
TOGETHER IN THAT SPACE, AGAIN,
THAT RISES THE TIDE FOR YOU ALL
BUT CERTAINLY MORE NIMBLE AND
MORE APPROPRIATE TO MEET PEOPLE
THAT WE WANT TO MAKE SURE HAVE
ACCESS.
>>BRIEFLY, IT'S VERY MUCH NOT A
BUYER'S MARKET FOR CLINICAL
TRIALS.
WHAT I MEAN, TRIALS WE BRING TO
CLINICAL RESEARCHERS AND
ULTIMATELY THE PATIENTS
THEMSELVES HAVE TO RESONATE WITH
PATIENT GROUPS.
IF WE'RE NOT CONNECTING IN WITH
NON-PROFITS, PATIENT ADVOCACY
GROUPS FROM THE VERY BEGINNING
TO OUTLINE WHAT TRIALS LOOK
LIKE, THE LIKELIHOOD OF THE
PRINCIPAL INVESTIGATOR AND
ULTIMATELY PATIENTS THEMSELVES
ACCEPTING THAT TRIAL IS LOW.
TO PUT A FINER POINT ON THAT, WE
THINK ABOUT THINGS LIKE
INCLUSION AND EXCLUSION CRITERIA
FOR CLINICAL TRIALS, THAT'S NOT
FOR US NECESSARILY TO HUDDLE IN
SOUTH SAN FRANCISCO OR BASAL
SWITZERLAND.
IT SHOULD BE DONE WITH PATIENT
ADVOCATE GROUPS.
FROM THE VERY BEGINNING OF OUR
CLINICAL TRIAL EFFORTS WE WANT
TO MIKE SURE WE'RE BRINGING
ALONG NON-PROFIT ORGANIZATIONS
TO REFINE STRATEGIES IN CLINICAL
DEVELOPMENT.
>>THANK YOU SO MUCH.
ALL RIGHT.
EARLIER WE DID PASS OUT INDEX
CARDS.
I DON'T KNOW WHO HAS FILLED THEM
OUT.
WE'RE GOING TO COLLECT THOSE AND
ASK DR. BENSON TO TAKE A LOOK
AND WHILE HE'S TAKING A LOOK AT
CARDS WE'LL ASK THE PANELISTS IF
THERE'S ANYTHING WE DIDN'T COVER
THAT YOU WANT TO CONVEY TO THE
AUDIENCE OR MAKE A TWO-MINUTE
APPEAL ABOUT WHATEVER IS ON YOUR
MIND WHILE HE GOES THROUGH THAT,
WE'LL DO FINAL COMMENTS AND GO
TO THE QUESTIONS.
>>FOR ME, I'VE HAD A COUPLE
CONVERSATIONS BUT THE IMPORTANCE
OF NOT ONLY REPRESENTING THE
PATIENT VOICE, ALLOWING PATIENTS
TO REPRESENT THEMSELVES,
CREATING SPACE AND DEMANDING IN
THE FORUMS THAT THERE'S
REPRESENTATION SPECIFICALLY FROM
GROUPS, MONUMENTALLY HELPFUL.
IT MEANS A LOT, GOES ON LONG WAY
TO UNDERSTAND HOW TO MAKE IT
REAL, IN ADDITION TO ADVOCACY
WORK.
I KNOW A LOT OF YOU DO THAT.
I APPLAUD YOU, BUT TO ENCOURAGE
THE WORK BECAUSE IT TAKES A LOT
TO MAKE SURE FOLKS ARE ABLE TO
ARTICULATE FOR THEMSELVES.
I'M GOING TO GIVE YOU A SLOGAN
THAT EVERYONE CAN USE.
WE KNOW THAT WE SPEND A LOT OF
TIME THINKING ABOUT HOW DO WE
EXPLAIN WHY PATIENTS SHOULD BE
INVOLVED IN CLINICAL TRIALS AND
COMMUNITY BENEFIT AND POTENTIAL
BENEFIT TO THE FAMILY, GOOD OF
THE ORDER, ALL THIS STUFF, ALL
TRUE.
LET'S REMEMBER YOU HEAR SLOGANS
FIRST PERSON CURED, PUT THE
DISEASE IN, OF ALZHEIMER'S, WILL
BE IN A CLINICAL TRIAL.
MAKE THAT A SLOGAN.
PEOPLE WOULD RESPOND.
THEY COULD BE THE FIRST ONE
THAT'S CLEARED OF DISEASE
BECAUSE THEY ARE PARTICIPATING
IN A CLINICAL TRIAL.
>>BRIEFLY I DON'T KNOW IF THIS
IS GOING COME AS A SURPRISE BUT
BIG PHARMA DOESN'T ALWAYS HAVE
THE BEST REPUTATION.
I WANT TO STRESS THE NOTION THAT
GOING FORWARD ONE OF OUR WAYS OF
BECOMING, IF YOU WILL, BETTER
STEWARDS AND BETTER PARTICIPANTS
IN CLINICAL DEVELOPMENT TO MAKE
SURE VOICES OF PATIENTS ARE
HEARD FROM THE ENTIRE DRUG
DEVELOPMENT PURPOSE, TO SAY THE
DOOR IS OPEN, WE WANT TO HEAR
FROM YOU, CONNECT, PARTNER AS WE
CONTINUE TO ADDRESS HEALTH
DISPARITIES, IT REQUIRES US TO
BE NIMBLE, OUR FINAL COMMENT,
THAT THE DOOR IS OPEN FOR
PARTNERSHIP.
>>I WANT TO TURN IT OVER TO MY
DIRECTOR, RICHARD BENSON.
>>THANK YOU.
WE HAD SEVERAL QUESTIONS HERE.
SOME HAVE BEEN ANSWERED THROUGH
YOUR CONVERSATIONS.
WE WILL -- AS DR. SANKAR
MENTIONED EARLIER WE GO GO
THROUGH THE QUESTIONS, AND THIS
IS PART OF AN ONGOING DISCUSSION
SO WE WILL ADDRESS ALL OF THE
QUESTIONS THAT HAVE COME TO US
AND INCORPORATE THAT INTO THE
THINGS WE'RE DOING MOVING
FORWARD.
ONE OF THE QUESTIONS, SEVERAL
QUESTIONS ACTUALLY DEAL WITH
RARE DISEASES.
AND COLLECTING DATA AND NOT
HAVING THE DATA.
I'D LIKE TO TIE THAT BACK TO
SORT OF THE EARLIER CONVERSATION
THAT WE HAD TODAY IN TERMS OF
THE DATA AND BIG DATA.
AND DISPARITIES CREATED THROUGH
MACHINE LEARNING AND A.I.
WHAT ARE YOUR CONVERSATIONS
YOU'RE HAVING NOW IN TERMS OF
DISPARITIES, BIG DATA, MACHINE
LEARNING, ARTIFICIAL
INTELLIGENCE?
>>I'LL START.
I'M OPTIMISTIC.
I SAY THAT BECAUSE ONE OF THE
SAVING GRACES THAT ALLOWS US TO
HAVE CERTAIN INSIGHT WHERE THE
PATIENTS ARE, WHAT THE
CAPABILITIES ARE, WHAT THE
IMAGING THAT'S AVAILABLE NEAR
THE PATIENTS AND SO FORTH SO WE
CAN BETTER REFINE WHERE AND HOW
WE DO CLINICAL RESEARCH.
SO FROM OUR PERSPECTIVE, I
RECOGNIZE ALL THE THREATS SPOKEN
TO BEFORE I CONTINUE TO FOCUS ON
THE PROMISE OF WHERE WE CAN USE
THESE VERY LARGE DATASETS TO
CONTINUE TO REFINE OUR CLINICAL
DEVELOPMENT STRATEGIES.
>>WE SHARE A LOT OF THE SAME
OPT MILES PER HOUR BUT --
OPTIMISM.
WE HAVE CONVERSATIONS ACROSS
ORGANIZATIONS TO MAKE SURE WE
DON'T EXACERBATE THE ISSUES
BECAUSE WE HAVEN'T BEEN AS
THOUGHTFUL SO WE HAVE A HEALTHY
SENSE I WOULDN'T SAY SKEPTICISM
BUT BEING CAUTIOUS AND
THOUGHTFUL IN THE WAY WE
LEVERAGE, PARTICULARLY AROUND
A.I.
>>VERY GOOD.
I'D LIKE TO GIVE A ROUND OF
APPLAUSE FOR THIS GREAT PANEL
AND THIS DISCUSSION.
I WANT TO THANK DR. THEODORE, WE
HAVE TWO LAWYERS HERE ON THIS
PANEL, ONE DOCTOR THE MINORITY,
ATTORNEY PEARSON AND ATTORNEY
MONROE, ALSO DR. SANKAR FOR THIS
EXCELLENT DISCUSSION THAT WE HAD
TODAY.
SOME OF THE THEMES HAVE COME UP
AS NAOMI MENTION AT THE
BEGINNING, TWO-YEAR STRATEGIC
PLAN PROCESS FOR HEALTH EQUITY
AND SO COMMUNITY ENGAGEMENT IS
SORT OF THE BIG PIECE OF THAT
THAT WE'RE LOOKING AT.
WE CAN HEAR THAT WITH ALL THE
PROGRAMS IN TERMS OF VARIOUS
UNIQUE WAYS, CREATING A PLAY AND
INVITING PEOPLE TO GIVE OUT
INFORMATION IN AN ENVIRONMENT
WHERE THEY FEEL COMFORTABLE
RECEIVING THAT INFORMATION.
OR CREATING A VAN TO GO INTO THE
COMMUNITY AND TO DO SCREENING
AND PASS OUT INFORMATION.
AND AS WELL AS THE ENGAGEMENT
ACTIVITIES THAT DR. THEODORE
MENTIONED AS WELL.
SO THIS IS THE KEY PIECE THAT WE
HEAR.
THAT'S IMPORTANT.
WE'RE THINKING OF RARE DISEASES,
EITHER DISEASES, VERY COMMON
DISEASE, IMPORTANT TO ENGAGE,
THAT'S WHAT WE'RE LOOKING TO DO
MOVING FORWARD AT NINDS.
AGAIN, IT WAS MENTIONED WE HAVE
A SERIES OF MANUSCRIPTS
PUBLISHED IN THE NEXT COUPLE
WEEKS IN NEUROLOGY.
WE HAVE THE SOCIAL DETERMINANTS
OF HEALTH FRAMEWORK, THE GOAL TO
SHOW THOSE AREAS OF INTERVENTION
WHERE RESEARCHERS CAN CONSIDER
WAYS OF ADDRESSING INEQUITY
PRIOR TO DEVELOPMENT OF DISEASE.
SO ACTUALLY THAT MANUSCRIPT, ONE
OF THE REVIEWERS, SEVERAL
REVIEWERS SAID THIS REQUIRED
READING FOR ANYONE INTERESTED IN
DOING BIOMEDICAL RESEARCH.
WE WANT THIS TO BE A GUIDE FOR
PEOPLE CONSIDERING DOING
RESEARCH IN THIS AREA AND TO
START THINKING ABOUT SOCIAL
DETERMINANTS OF HEALTH.
WE'RE EXCITED ABOUT THIS.
AGAIN, THIS IS THE FIRST PART OF
OUR DISCUSSION.
I'M GOING TO NOW TURN IT OVER TO
MY INSTITUTE DIRECTOR WHO HAS
BEEN SUPPORTIVE OF THE WORK
WE'RE DOING.
WE'VE DONE A LOT OF NOVEL THINGS
IN THIS SPACE.
THANK YOU.
DR. KOROSHETZ HAS BEEN
SUPPORTIVE OF THE WORK.
HE AND I HAD AN OPPORTUNITY TO
PUBLISH AN ARTICLE TOGETHER IN
STROKE, IN TERMS OF HEALTH
EQUITY RESEARCH THAT MATTERS.
SO WE FOCUS ON DOING THE TYPE OF
WORK THAT WILL ACTUALLY BE
TRANSFORMATIVE HOW WORK IS DONE.
THAT'S WHAT WE'RE MOVING FORWARD
WITH DOING AT NINDS.
DR. KOROSHETZ?
>>OKAY.
WELL, I HAVE THE PLEASURE OF
WRAPPING UP THE NON-PROFIT
FORUM, WHICH I MUST SAY, I'LL
SAY WHAT I SAY EVERY YEAR, WOW,
THIS IS BETTER THAN LAST YEAR.
THAT'S BECAUSE OF ALL THE PEOPLE
THAT DID A LOT OF HARD WORK
TRYING TO THINK HARD ABOUT HOW
TO MAKE THIS VALUABLE, TO THE
FOLKS WHO COME EACH YEAR TO THE
NINDS FOLKS, AND PEOPLE FROM
OTHER PARTS OF THE GOVERNMENT
AND INDUSTRY.
AND SO IT'S ALL PUT TOGETHER BY
A STEERING COMMITTEE OF PEOPLE
FROM NON-PROFITS WORKING WITH
NIH FOLKS, AND THEY SPEND ABOUT
A YEAR TRYING TO THROW THINGS
OUT AGAINST THE WALL, SEE WHAT
STICKS, AND EVERY YEAR THEY DO A
GREAT JOB AND THEY ALWAYS TRY
AND DO BETTER THAN THE YEAR
BEFORE AND ALWAYS SUCCEED.
NEXT YEAR I'M SURE THEY WILL AS
WELL.
BUT IT IS A LOT OF WORK.
AND WE SAW THE FRUITS OF THEIR
LABOR OVER THE LAST TWO DAYS.
A COUPLE REFLECTIONS ON MY END
THAT, YOU KNOW, WE ARE ALL
INVOLVED IN THE SAME ECOSYSTEM
IN A DIFFERENT WAY.
AND THE GOAL THAT BINDS US
TOGETHER IS TO FIND BETTER
TREATMENTS FOR PEOPLE WITH
NEUROLOGIC DISEASES THAT REALLY
TRAGIC, AS YOU HEARD YESTERDAY,
AND AS WE ALL KNOW, FROM GROUPS
YOU TAKE CARE OF AND REPRESENT,
AND THIS ECOSYSTEM AT THIS
MEETING HAS SHIFTED TOWARDS
PATIENT ADVOCACY GROUPS AND HOW
THEY CAN WORK WITH
INVESTIGATORS, HOW THEY CAN WORK
WITH NINDS.
WE HAVE MANY OTHER MEETINGS
THROUGHOUT THE YEAR WHERE WE
MIGHT WORK WITH SCIENTISTS MORE,
BUT AS AMY SAID WE'RE REALLY
INTERESTED IN NOT DOING THESE
THINGS SEPARATELY BUT WEAVING
THE ECOSYSTEM TOGETHER AS WE DO
THIS.
WE ALL ARE BASICALLY -- THE
NON-PROFITS AND WE ARE BASICALLY
INTERACTING WITH THE SCIENTISTS
AND PATIENTS, AND THE
CAREGIVERS.
AND WE ARE ALL PUSHING TO GET
MORE WORK DONE.
I DID WANT TO SAY ONE THING THAT
I THINK IS SOMETHING THAT WE
HAVEN'T MENTIONED BEFORE.
WE KEEP TALKING ABOUT, YOU KNOW,
THE INVESTIGATORS AS BEING THE
P.I.s OF THESE PROJECTS, OUR
MONEY GOES OUT TO THE PRINCIPAL
INVESTIGATORS OF THESE PROJECTS.
BUT THE PEOPLE WHO REALLY DO THE
WORK ON THE PROJECTS WE TEND TO
IGNORE.
MAYBE THAT'S SOMETHING THAT WE
COULD THINK ABOUT HOW TO
ADDRESS.
I CAN TELL YOU NOW THAT MANY OF
THE PROJECTS THAT WE'RE FUNDING,
THE REASON WHY THINGS ARE NOT
GOING WELL IS BECAUSE THEY DON'T
HAVE CLINICAL COORDINATORS OFTEN
POSTBACS OR NURSES, DON'T GET IN
THE FIELD, CAN'T STAY IN THE
FIELD, MAYBE NOT PAID WELL
ENOUGH OR APPRECIATED ENOUGH.
BUT I WOULD SAY THOSE ARE THE
PEOPLE WHO REALLY THE DRIVERS OF
WHAT HAPPENS ON THE GROUND.
MAYBE WE CAN PAY SOME ATTENTION
TO THAT COMING UP BECAUSE IT IS
ONE OF THE ISSUES.
THE OTHER ISSUE THAT PEOPLE MAY
NOT KNOW ABOUT IS THAT, AGAIN,
IN THE LABORATORIES THE WORK IS
DONE PRIMARILY BY IN OUR
AMERICAN SYSTEM BY POSTDOCS,
PEOPLE WHO COME IN, FINISH THEIR
TRAINING, IN TERMS OF GETTING A
Ph.D., NOW GETTING
PROFESSIONALIZED EXPERIENCE,
HOPEFULLY GOING TO COMPETE FOR
THEIR OWN LABORATORY OR
POTENTIALLY MOVING TO A
DIFFERENT AREA OF SCIENCE
MANAGEMENT.
BUT THERE'S A BIG SHORTAGE IN
THAT SPACE NOW TOO.
THE NIH HAS AN ADVISORY
COMMITTEE TO THE DIRECTOR THAT'S
LOOKING INTO THE ISSUE OF
POSTDOC POSITIONS IN THE U.S.,
BUT, AGAIN, THAT'S ANOTHER WEAK
LINK IN THE CHAIN THAT WE
SOMETIMES DON'T THINK TOO WELL
ABOUT.
NON-PROFITS THOUGH I THINK CAN
HELP IN THAT SPACE, THE DISEASE
YOU FOCUS ON, POTENTIALLY GIVING
THEM SOME FUNDING TO GET THEIR
PROGRAMS STARTED AS THEY CAN
THEN TAKE OUT AND COMPETE FOR
NIH FUNDING.
I SAY EVERY YEAR THAT I WOULD
URGE THE GROUPS TO THINK ABOUT
HOW YOUR FUNDING CAN BE USED TO
LEVERAGE OUR FUNDING.
SO AS I MENTIONED, IN MY TALK
YESTERDAY, OUR SYSTEM OF GIVING
OUT FUNDS IS A COMPETITIVE ONE,
PEOPLE COME IN, GET THE SCORE,
BEST SCORES GET FUNDED.
ONE OF THE THINGS TO DETERMINE
THE BEST SCORE HOW MANY
PRELIMINARY DATA, THERE WE DON'T
HAVE A GOOD SYSTEM FOR GIVING
PEOPLE MONEY TO GET PRELIMINARY
DATA.
MOST OF THAT COMES FROM THE
NON-PROFITS, IN MY EXPERIENCE.
SO THAT'S ANOTHER REALLY AMAZING
TOOL.
YOU SHOULD KEEP TRACK OF THAT,
PEOPLE YOU FUND, HOW SUCCESSFUL
ARE THEY, TO LEVERAGE NIH GRANT.
WHICH THE AVERAGE COST RIGHT
NOW, TOTAL COST IS ABOUT
$550,000 A YEAR.
SO REALLY QUITE EXPENSIVE GRANTS
THAT PEOPLE CAN GET FROM THE
NIH.
AND THEN WE HEARD YESTERDAY
ABOUT, YOU KNOW, THIS NEW REALLY
NEW WORLD OF TREATMENT, GENOMIC
THERAPY.
AND THAT, YOU KNOW, IN CONCERT
WITH THE MOVE IN NEUROLOGY,
NEUROSURGERY TOWARDS MORE OF A
TREATMENT, YOU KNOW, USED TO BE
WHEN -- I REMEMBER WHEN I WAS
LOOKING AT NEUROLOGY I DECIDED
I'M NOT GOING TO DO NEUROLOGY,
NO TREATMENTSES, I WANT TO TREAT
PEOPLE, CHANGED MY MIND.
I WAS GLAD I DID BECAUSE
TREATMENTS STARTED COMING IN MY
CAREER, IT'S BEEN EXCITING.
WE'RE GETTING MORE AND MORE INTO
THAT SPACE.
BUT TREATMENTS ARE NOT WITHOUT
RISK, AND RESEARCH RELIES ON
PEOPLE WHO ENTER RESEARCH
PROGRAMS AND PUT THEMSELVES ON
THE LINE.
I THINK MARIA SAID IT BEST
YESTERDAY THAT YOU DON'T ENTER
RESEARCH BECAUSE IT'S GOING TO
HELP YOU.
YOU THINK IT'S GOING TO HELP
SOMEBODY ELSE, EITHER IN YOUR
LIFETIME OR MAYBE NOT IN YOUR
LIFETIME.
SO IT'S REALLY ALTRUISM THAT WE
RELY ON FOR OUR RESEARCH
INFRASTRUCTURE FOR PEOPLE TO
PARTICIPATE, SUBJECTS TO
PARTICIPATE.
AND WHY THEY DO THAT, YOU KNOW,
BECAUSE THEY ARE REALLY
INVESTED, THEY REALLY FEEL LIKE
THEY CAN CONTRIBUTE AND
CERTAINLY CAN.
THEY ARE ONLY GOING TO
CONTRIBUTE IF THEY TRUST THE
SYSTEM.
HOW DO YOU BUILD TRUST?
THE GOVERNMENT IS NOT GOING TO
BE THE ANSWER TO BUILDING TRUST.
SO, WHO IS GOING TO DO IT?
I DON'T THINK THE COMPANIES ARE
GOING TO DO IT.
I THINK THEY CAN HELP BUT THEY
HAVE THEIR AGENDAS.
EVERYBODY KNOWS WHAT THEY ARE.
BUT THE NON-PROFITS, THAT'S
WHERE THE TRUST IS.
THAT TRUST CAN SPREAD OUT TO THE
GOVERNMENT, TO THE INDUSTRY, BUT
WITHOUT THE NON-PROFITS I THINK
WE'RE ALL IN BIG TROUBLE.
BUILDING TRUST IN THE ECOSYSTEM
FOR RESEARCH IS NOT GOING TO
HAPPEN WITHOUT YOU.
SO I MUST SAY WE OWE YOU A REAL
DEBT OF GRATITUDE FOR WHAT YOU
DO.
WE ALSO HEARD ABOUT THE SUCCESS
STORIES AND AS I SAID TO HAVE
THREE DRUGS APPROVED FOR OUR
INDICATIONS, THESE ARE REALLY
SERIOUS DISEASES.
THEY ARE REALLY BREAKTHROUGHS IN
THESE SPACES.
WHAT WE'LL SEE IN ALZHEIMER'S IS
JUST ASTRONOMICAL CHANGE IN HOW
HEALTH CARE SYSTEMS WILL HAVE TO
ADAPT TO THAT.
AND I THINK WE'RE LOOKING
FORWARD TO MORE OF THESE.
THESE ARE THE BEST.
THAT'S WHAT EVERYBODY IS
STRIVING FOR.
AS YOU CAN SEE IT TAKES A LONG
TIME TO GET THERE.
THEY JUST HAVE TO GET INTO THE
LONG RUN.
BUT I THINK THAT FUTURE DOES
LOOK BRIGHT IF WE CAN WORK
TOGETHER AND STICK TOGETHER FOR
THE LONG RUN HERE.
LAST, WE HEARD ABOUT DATA
SHARING.
AND I THINK IT WAS A REALLY GOOD
CONVERSATION.
WE HAD ANOTHER SIMILAR
CONVERSATION WITH THE BRAIN
NEUROETHICS WORKING GROUP LAST
WEEK, AND I THINK THAT AS RON
SAID, THE PATIENT DATA IS
REALLY, REALLY IMPORTANT.
YOU HAVE TO BE REALLY CAREFUL
THAT YOU'RE COLLECTING DATA
THAT'S GOING TO BE USEFUL.
SO THERE IS, AND I CAN SEE IT
FROM WHERE I STAND, WITH
GENERAL, YOU KNOW, THING ABOUT
LET'S COLLECT A LOT OF DATA AND
THEN FIGURE OUT WHAT IT'S GOOD
FOR LATER.
THAT USUALLY DOESN'T WORK.
USUALLY WHEN YOU TRY TO FIND OUT
WHAT YOU'RE GOING TO USE IT FOR
YOU REALIZE, GEE, WE DIDN'T
COLLECT THIS.
WITHOUT COLLECTING THAT, NONE OF
THE OTHER STUFF IS VERY GOOD.
YOU HAVE TO BE CAREFUL ABOUT HOW
YOU DESIGN WHAT KIND OF DATA
YOU'RE GOING TO TAKE IN.
IF YOU DO IT RIGHT, IT'S
INCREDIBLY VALUABLE.
AT THE NINDS, RON TALKED ABOUT
NATURAL HISTORY DATA, 400
DISEASES, WE COULD POTENTIALLY
USE ALL THE MONEY TO FUND JUST
NATURAL HISTORY DATA.
WE PURPOSELY DON'T DO THAT.
WE TEND TO FOCUS ON WHAT WE CALL
CLINICAL TRIAL READINESS.
SO YOU HAVE A DISEASE, WHERE
THINGS LOOK LIKE THEY ARE GOING
TO BREAK, YOU NEED TO DO A STUDY
THAT'S GOING TO GET YOU THE
OUTCOME MEASURES YOU NEED FOR
YOUR TRIAL OR BIOMEDICAL YOU
NEED FOR YOUR TRIAL.
IT'S MUCH MORE FOCUSED TOWARDS
THAT ASPECT.
THEN WE HAVE PROGRAMS FOR THAT.
BUT NON-PROFITS DO A LOT OF
BACKGROUND COLLECTION OF NATURAL
HISTORY BECAUSE IT'S JUST TOO
OVERWHELMING GIVEN THE NUMBER OF
DISORDERS WE HAVE.
THE OTHER THING WHICH WAS
TOUCHED UPON IN TODAY'S SESSION
BUT WAS MORE DISCUSSED AT THE
BRAIN DATA, THAT WITH THE
TECHNOLOGIES COMING ON NOW
PEOPLE PUT IN THEIR DATA, AND
THEY DO IT FOR PARTICULAR
PURPOSE, WHEN THEY FIRST START.
BUT THERE'S ALSO THE LIKELIHOOD
THAT THAT DATA IS GOING TO BE
USED YEARS LATER FOR THINGS THAT
THEY HAD NO IDEA ABOUT.
A BIG ETHICAL ISSUE, YOU SUBMIT
YOUR DATA FOR CERTAIN PROJECTS,
YOU KNOW, WHAT IS THE CONSENT
PROCESS THAT YOU AGREE TO HAVE
YOUR DATA USED FOR SOMETHING
ELSE.
WHICH YOU MAY NOT EVEN BE ABLE
TO GUESS AT.
LOTS OF ISSUES THAT FACE US.
BUT REALLY FROM THE MEETING
YESTERDAY AND TODAY A LOT OF
PROGRESS GOING ON.
AND STILL THINGS WE NEED TO WORK
OUT, PARTICULARLY ON THE HEALTH
EQUITY SIDE AS YOU HEARD HERE
TODAY.
THE ALZHEIMER'S ISSUE IS THE
REAL LESSON BECAUSE WE DON'T
HAVE THE DATA TO KNOW WHAT THE
ANTI-AM AMYLOID THERAPY IS GOINO
DO IN THE AFRICAN AMERICAN
POPULATION.
WE KNOW FROM THE BEGINNING THAT
AFRICAN AMERICANS WHO HAVE
DIAGNOSIS OF ALZHEIMER'S HAVE
LESS AMYLOID THAN CAUCASIANS.
WHAT DOES THAT MEAN FOR THE
THERAPY TO TAKE AMYLOID OUT?
APOE 4 LESS OF A DRIVER IN
ALZHEIMER'S IN THE AFRICAN
AMERICAN GROUP TURNS OUT TO BE A
MARKER THAT MAY BE
CONTRAINDICATION TO GETTING
THESE DRUGS IN THE CAUCASIAN
GROUP BUT WE HAVE NO IDEA IF THE
DRUG WORKS IN AFRICAN AMERICANS.
SO JUST AN EXAMPLE OF HOW WE GET
BURNED WHEN WE DON'T HAVE THE
KIND OF DATA WE NEED FROM
DIFFERENT POPULATIONS IN THE
U.S.
SO, I THINK WE HEARD A LOT OF
CHALLENGES AND SUCCESSES.
LOOKING FORWARD TO NEXT YEAR
ALREADY.
CAN'T WAIT.
THANKS VERY MUCH.