>> JILL: WELCOME, EVERYONE TO
SESSION SIX OF ETHICAL AND
REGULATORY ASPECTS OF CLINICAL
RESEARCH. IT'S HARD TO IMAGINE
WE JUST HAVE ONE MORE SESSION.
TODAY, WE'RE GOING TO HAVE, I'M
GOING TO INTRODUCE DR. FISHER IN
A MOMENT SHE'S GOING TO GIVE A
PRESENTATION THEN WE'RE GOING TO
WORK THROUGH A CASE WITH ME AND
THEN WE'RE GOING TO HAVE A PANEL
OF INDIVIDUALS WHO HAVE
PARTICIPATED IN CLINICAL TRIALS
HERE AT THE CLINICAL CENTER.
I'M VERY EXCITED ABOUT THAT.
THAT WILL BE AFTER OUR BREAK.
AND ONE QUICK, TWO QUICK
ANNOUNCEMENTS. IF YOU HAVE
QUESTIONS, PLEASE POST THEM IN
THE DISCUSSION BOARD OR SEND
THEM TO THE E-MAIL, AND FOR
THOSE OF YOU INTERESTED IN WHEN
YOU WILL RECEIVE YOUR
CERTIFICATES, THE COURSE CLOSES
ON NOVEMBER 20TH WHICH MEANS
THAT ANYONE WHO NEEDS TO CATCH
UP ON VIDEOS AND QUIZZES CAN DO
SO. WE ARE GOING TO SEND THE
CERTIFICATES WITHIN TWO WEEKS
AFTER THAT, SO IT'LL BE A LITTLE
WHILE BUT YOU'LL DEFINITELY GET
ONE. SO WITH THAT, I'M GOING TO
INTRODUCE DR. JILL FISHER
JOINING US FROM THE DEPARTMENT
OF SOCIAL MEDICINE AND THE
CENTER FOR BIOETHICS AT THE
UNIVERSITY OF NORTH CAROLINA.
SHE HOLDS A PH.D. IN SCIENCE AND
TECHNOLOGY STUDIES AND
EXCITINGLY FOR US, HER RESEARCH
FOCUSES ON HOW CLINICAL TRIALS
ARE CONDUCTED AND WHO
PARTICIPATES IN THEM AS
RESEARCHERS AND PARTICIPANTS AND
TODAY SHE'S GOING TO TALK WITH
US ABOUT HEALTHY PARTICIPANTS IN
CLINICAL TRIALS.
>> JILL: THANK YOU SO MUCH. I'M
REALLY EXCITED TO BE HERE TODAY.
THERE'S A LOT THAT I COULD SAY
ABOUT HEALTHY VOLUNTEERS AND I
WILL SAY A LOT. BUT I THOUGHT
IN PARTICULAR I WOULD FOCUS ON
ETHICAL ISSUES RELATED TO
RESEARCH PAYMENTS SINCE THIS IS
ONE OF THE AREAS THAT MAKES
HEALTHY VOLUNTEER TRIALS
DIFFERENT FROM CLINICAL TRIALS
WITH PATIENTS. SO AS I
UNDERSTAND IT YOU'VE ALREADY
GOTTEN A LITTLE BIT OF A PRIMER
ON DRUG DEVELOPMENT AND HOW
DRUGS GET TO THE MARKET AND WHAT
THE WHOLE PROCESS IS LIKE BUT I
THOUGHT I WOULD REVIEW THIS TO
COMPLICATE THE IDEA OF HOW THIS
PROCESS WORKS A LITTLE BIT. SO
USUALLY WHEN WE THINK ABOUT DRUG
DEVELOPMENT IT'S DEPICTED AS A
VERY LINEAR PROCESS OFTENTIMES
WITH AN ARROW OR A FUNNEL OR
SOMETHING LIKE THAT WHERE WE SEE
ON ONE END WE START WITH BASIC
RESEARCH AND IF THINGS LOOK
PROMISING IT MIGHT MOVE INTO
PRECLINICAL ANIMAL RESEARCH AND
THEN IF THAT LOOKS PROMISING WE
WOULD START HUMAN TRIALS AND WE
HAVE THIS SYSTEM IN THE U.S. OF
THINKING ABOUT CLINICAL TRIALS
AS PHASED SO ONE, TWO, AND
THREE. AND SO FOR THE PURPOSES
OF THE FDA, YOU KNOW, IF WE
ACTUALLY WANT TO GET A DRUG OR
THERAPY ON THE MARKET, DRUGS
HAVE TO GO THROUGH THESE STEPS
SO PHASE ONE TRIALS ARE THE ONES
THAT ARE DESIGNED TO EVALUATE
THE SAFETY AND TOLERABILITY OF
AN INVESTIGATIONAL PRODUCT.
THESE ARE THE ONES THAT USUALLY
USE HEALTHY VOLUNTEERS. AS LONG
AS THERE AREN'T ANY MAJOR SAFETY
ISSUES THAT EMERGE FROM THERE.
THE STUDIES MIGHT PROCEED INTO
PHASE TO TRIALS AND I LIKE TO
THINK OF THESE AS PROOF OF
CONCEPT STUDIES, THESE ARE
RELATIVELY SMALL, NOW THEY'RE
ACTUALLY USING PATIENTS AS THEIR
PARTICIPANT POPULATION BUT
THEY'RE STILL LOOKING AT SAFETY
AND ISSUES AT THE SAME TIME AS
DRUG I HAVE -- EFFICACY
IF SO, THEN IT WILL PROCEED TO
PHASE THREE TRIALS WHICH ARE
MORE THE ONES WE TEND TO THINK
OF BECAUSE THESE ARE THE LARGER
SCALE ONES, THESE ADHERE TO
COMPLETE AND THE ONES THAT FDA
IS BASED ON. I WANT TO
COMPLICATE THIS NOTION A LITTLE
BIT BECAUSE IT DOES IMPLY THAT
ONE STEP HAS TO OCCUR TO GET TO
THE NEXT STEP AND THAT IT'S ALL
DONE IN THIS VERY ORDERLY
FASHION BUT WHEN WE ACTUALLY
LOOK AT THE CLINICAL TRIAL
PORTFOLIOS, OF PHARMACEUTICAL
COMPANIES WE SEE IT'S ACTUALLY
MUCH MORE COMPLICATED THAN THAT
AND MUCH MORE FLUID. SO FOR
INSTANCE, FOR THE WHOLE PROCESS
OF NON-CLINICAL RESEARCH,
NONHUMAN ANIMAL STUDIES
CONTINUE. SO THESE ARE
CONTINUING OFTENTIMES RIGHT UP
TO FDA APPROVAL SO HUMAN TRIAL
CONSIST START BEFORE THE ANIMAL
STUDIES ARE DONE AND SO I
MENTION THIS BECAUSE ONE OF THE
REASONS THAT THIS IS IMPORTANT
IS A LOT OF TIMES THERE ISN'T
VERY MUCH KNOWN ABOUT
REPRODUCTIVE TOXICITY WHEN THEN
HAS IMPLICATIONS FOR WHO IS
ALLOWED TO ENROLL IN EARLY PHASE
HUMAN TRIALS. ANOTHER IMPORTANT
PIECE OF THIS IS THAT WHEN WE
THINK ABOUT HEALTHY VOLUNTEERS
IN THIS DRUG DEVELOPMENT PROCESS
WE OFTEN THINK OF THEM AS THE
ONES THAT ARE DOING THESE FIRST
IN HUMAN STUDIES SO LITERALLY
THE FIRST TIME THAT A HUMAN IS
TAKING A DRUG OR TREATMENT.
HOWEVER THERE ARE OTHER STUDIES
THAT ARE LOOKING AT SAFETY,
TOLERABILITY AND THESE ACTUALLY
CONTINUE UP TO SOMETIMES THE
TIME A COMPANY IS SUBMITTING
THEIR NEW DRUG APPLICATION TO
THE FDA. I WILL TALK, YEAH SO,
IN ADDITION TO THOSE FIRST IN
MAN OR FIRST IN HUMAN STUDIES
THAT ARE REALLY, AGAIN, YOU
KNOW, SOMETIMES WE THINK OF IT
AS AN A, PHASE ONE TRIAL BUT
REALLY IS A WHOLE PORTFOLIO OF
CLINICAL TRIALS BECAUSE WHAT'S
HAPPENING IS IT REQUIRES
MULTIPLE STUDIES TO MEASURE
TOLERABILITY SO YOU SEE
INCREASES DOSES OF A DRUG AND
WHAT HAPPENS WHEN YOU GIVE THEM
TO HEALTHY VOLUNTEERS.
SOMETIMES MULTIPLE DOSES. SO
ALL OF THESE THINGS ARE PART OF
THE PHASE ONE PROCESS. BUT THEN
AS I WAS SAYING, MAYBE LATER IN
THE DRUG DEVELOPMENT PROCESS
WHEN A COMPANY BELIEVES THAT A
DRUG MIGHT ACTUALLY GET TO THE
MARKET THEY ARE INCENTIVIZED TO
DO OTHER KINDS OF STUDIES AND
THEY USE HEALTHY VOLUNTEERS FOR
THESE TOO. IF YOU THINK ABOUT
PRESCRIPTION DRUGS THAT YOU MAY
HAVE TAKEN AT VARIOUS POINTS IN
YOUR LIFE, IT MIGHT SAY TAKE
THIS DRUG ON AN EMPTY STOMACH OR
WITH A MEAL SO THEY USE THOSE
WITH VOLUNTEERS. ADDITIONALLY
SOMETIMES THE FDA REQUIRES
COMPANIES TO DO DRUG DRUG
INTERACTION STUDIES BECAUSE THEY
KNOW PATIENT POPULATIONS MIGHT
BE TAKING THIS NEW DRUG.
SO HEALTHY VOLUNTEERS ARE ALSO
USED FOR INTERACTION STUDIES AND
A HUGE USE OF HEALTHY VOLUNTEERS
IS TO GET GENERIC DRUGS ON THE
MARKET SO TO GET A GENERIC ON
THE MARKET A COMPANY DOESN'T
NEED TO GO THROUGH THE WHOLE
PROCESS OF RETESTING THE DRUG IN
THE SAME SENSE AS THE BRAND NAME
DRUG. THEY JUST NEED TO PROVE
THAT IT'S BIOEQUIVALENT TO THAT
BRAND NAME DRUG SO IN OTHER
WORDS THAT THE DRUG IS ABSORBED,
METABOLIZED, DISTRIBUTED AND
EXCRETED IN THE SAME WAY THAT
THE BRAND NAME DRUG SO THEY USE
HEALTHY VOLUNTEERS FOR THESE
STUDIES THIS IS TO SAY THAT
HEALTHY VOLUNTEERS ARE USED IN
LOTS OF DIFFERENT KINDS OF
STUDIES AS PART OF THE DRUG
DEVELOPMENT PROCESS BUT FOCUSED
ON SAFETY AND TOLERABILITY. SO
WHY USE HEALTHY VOLUNTEERS? WHY
NOT JUST USE PATIENTS SO THERE
ARE A VARIETY OF REASONS THAT
HEALTHY VOLUNTEERS ARE USED IN
CLINICAL RESEARCH. SO, ONE
RATIONALE IS THE ONE THAT I
THINK OF AS SEPARATING THE
SIGNAL FROM THE NOISE IF YOU USE
PATIENTS AND YOU'RE TRYING TO
DETERMINE THE SAFETY PROFILE OF
A DRUG IT COULD BE DIFFICULT TO
DETERMINE WHAT IS -- WHAT KIND
OF SYMPTOMS ARE CAUSED BY THE
DRUG ITSELF VERSUS WHAT'S KIND
OF A SYMPTOM RELATED TO THE
ILLNESS ITSELF SO HEALTHY
VOLUNTEERS ARE SORT OF A CLEAN
SLATE IN THIS SENSE SO
POTENTIALLY TO HELP ADJUDICATE
WHETHER SOMETHING IS A DRUG
AFFECT OR NOT. IT'S ALSO BEEN
ARGUE THAT HAD BECAUSE HEALTHY
VOLUNTEERS ARE HEALTHY THERE'S
LESS RISK TO THEM OF TAKING AN
INVESTIGATIONAL DRUG SO IF WE
THINK ABOUT THEIR KIDNEYS AND
LIVERS THESE ARE FUNCTIONING
NORMALLY SO WE WOULD EXPECT THEM
TO PROCESS DRUGS MORE QUICKLY
AND CLEAR THEM OUT OF THEIR
SYSTEM. THAT'S ANOTHER REASON
WHEN WE LOOK AT SAFETY ISSUES TO
USE HEALTHY VOLUNTEERS. ANOTHER
BIG REASON WHICH IS, I KIND OF
THINK OF AS PROBABLY THE BIGGEST
REASON DRIVING THIS IS THAT
HEALTHY VOLUNTEERS ARE MUCH MORE
AVAILABLE THAN PATIENTS ARE. SO
THEY'RE SORT OF THIS SPEED AND
ECONOMIC REASON TO USE HEALTHY
VOLUNTEERS THAT IT'S MUCH
QUICKER AND EASIER TO CREATE A
COHORT OR A PANEL OF HEALTHY
VOLUNTEERS TO DO THESE CLINICAL
TRIALS THAN IT WOULD BE TO TRY
TO FIND PATIENTS TO DO THEM.
AND THEN FINALLY, THERE'S ALSO
AN ETHICAL RATIONALE THAT'S
GIVEN IN THE SENSE THAT WE DON'T
TYPICALLY EXPECT THERE TO BE ANY
BENEFIT TO PARTICIPANTS IN PHASE
ONE OR EARLY PHASE TRIALS. AND
SO GIVEN THAT WE DON'T EXPECT
THAT BENEFIT, THERE'S THIS IDEA
THAT WHY DON'T WE SAVE PATIENTS
FOR TRIALS THAT MIGHT ACTUALLY
BENEFIT THEM MORE SO PHASE TWO
OR PHASE THREE TRIALS. TO BE
ABLE TO CONVEY THAT BENEFIT TO
PARTICIPANTS. SO A REALLY
UNIQUE ASPECT OF HOW PHASE ONE
TRIALS ARE CONDUCTED TO PHASE
THREE TRIALS IS THAT THEY'RE
USUALLY CONDUCTED AS INPATIENT
CONFINEMENT STUDIES AND THAT
MEANS THAT PARTICIPANTS ARE
LITERALLY CHECKING INTO A
RESEARCH FACILITY FOR THE LENGTH
OF THE TRIAL. AND THE LENGTH
COULD VARY DRAMATICALLY
DEPENDING ON THE DESIGN OF THE
STUDY. SOMETIMES IT'S JUST A
DAY OR TWO. ONE OF THE STUDIES
THAT PARTICIPANTS THAT WERE IN
MY STUDY OF HEALTHY VOLUNTEERS
WE'RE IN WAS A 45 DAY STUDY SO
THEY LITERALLY CHECKED INTO A
RESEARCH FACILITY AND DIDN'T
LEAVE IT FOR 45 DAYS YOU CAN
IMAGINE TO GET PEOPLE TO DO THIS
YOU HAVE TO PAY THEM OTHERWISE
IT WOULD BE A LITTLE BIT HARD TO
IMAGINE THAT PEOPLE WOULD GIVE
UP SO MUCH OF THEIR TIME TO DO
IT. AND SO THE TYPICAL
COMPENSATION FOR THESE TRIALS IS
BASED ON THE LENGTH OF THE TRIAL
AND SPECIFICALLY HOW MANY NIGHTS
THEY HAVE TO SPEND IN THE
FACILITY SO THE GOING RATE IS
ABOUT TWO HUNDRED TO TWO HUNDRED
FIFTY DOLLARS A NIGHT. AND THE
AVERAGE CLINICAL TRIAL PAYS
ABOUT 3,000 DOLLARS. ANOTHER
COMPONENT OF THIS IS THAT THE
RESEARCH CLINICS REALLY TEND TO
LIKE TO USE PARTICIPANTS WHO
HAVE DONE THE STUDIES BEFORE.
BECAUSE THEY NEED TO STAY IN
THESE PLACES AND OFTEN PEOPLE
HAVE THE RIGHT TO WITHDRAW FROM
CLINICAL RESEARCH. THE HOPE IS
THAT THEY WON'T WITHDRAW AND SO
A LOT OF TIMES THE RESEARCH
FACILITIES FEEL LIKE WELL, IF WE
HAVE PEOPLE THAT WE KNOW AND WE
TRUST WE'LL ACTUALLY GET THROUGH
THE STUDIES. THAT'S BENEFICIAL
TO US. SO THEY OFTEN LOOK FOR
PEOPLE WHO HAVE GONE THROUGH
TRIALS BEFORE, KNOW WHAT IS PART
OF THOSE TRIALS. AND PRIORITIZE
THOSE INDIVIDUALS. AND I'LL
TALK ABOUT THE PEOPLE WHO WANT
TO PARTICIPATE OVER AND OVER
AGAIN IN STUDIES AS WELL. IT
OFTEN, PEOPLE OFTEN ASK ME,
LIKE, WELL HOW RISKY ARE THESE
STUDIES TO HEALTHY VOLUNTEERS
AND THAT'S A HARD QUESTION TO
UNDERSTAND. OF COURSE IT
DEPENDS ON WHAT THE
INVESTIGATIONAL DRUG IS AND WHAT
PROCEDURES ARE PART OF A STUDY
AS WELL. SOME PEOPLE HAVE TRIED
TO QUANTIFY THIS BY DOING
METAANALYSES OF MULTIPLE STUDIES
TO CREATE A PICTURE OF PHASE ONE
TRIALS. AND SO THOSE ANALYSES
SHOW THAT ABOUT 65% OF ALL
HEALTHY VOLUNTEERS ARE GOING TO
EXPERIENCE AT LEAST ONE ADVERSE
EVENT DURING A CLINICAL TRIAL.
THIS COULD BE SOMETHING VERY
MILD LIKE MAYBE A MILD HEADACHE,
MAYBE GASTROINTESTINAL CHANGES
AND THINGS LIKE THAT AND THEY
TEND TO BE TRANSIENT AND THEIR
QUICK TO RESOLVE. THAT SAID
ABOUT 1-4% OF ADVERSE EVENTS ARE
SERIOUS. AND I THINK PROBABLY
MANY OF YOU HAVE HEARD OF SOME
OF THE PHASE ONE CATASTROPHES
THAT HAVE HAPPENED SO THERE WAS
A DEATH AT HOP KINS IN 2001.
THERE WAS A DEATH AT LILY
TESTING FACILITY IN 2004.
PROBABLY THE CASE WE TALK ABOUT
THE MOST IS THE INJURY IN LONDON
IN 2006. THIS WAS WHEN A
BIOLOGIC WAS GIVEN AND THE
PARTICIPANT WHO IS RECEIVED THE
ACTIVE DRUG WENT INTO MULTIPLE
ORGAN FAILURE VERY QUICKLY AFTER
RECEIVING THE DRUG.
AND IT RAISED QUESTIONS ABOUT
THE TIMING OF DOSING HEALTHY
VOLUNTEERS. AND THEN MOST
RECENTLY THERE WAS A DEATH AT
THE BIO TRIAL FACILITY IN FRANCE
IN 2016.
IT'S RARE THAT SOMEONE WILL DIE
FROM A PHASE ONE TRIAL BUT IT
DOES HAPPEN. I BECAME REALLY
INTERESTED IN THE PHASE ONE
INDUSTRY WHEN I WAS DOING
ANOTHER PROJECT THAT WAS REALLY
LOOKING AT THE OUTSOURCING OF
PHARMACEUTICAL CLINICAL TRIALS
AWAY FROM ACADEMIC MEDICAL
CENTERS TO FOR-PROFIT COMPANIES
THAT WERE CONDUCTING THEM AND AS
I WAS DOING THAT RESEARCH WHEN I
STARTED TO SEE WAS ALL THESE
FACILITIES WERE BEING BUILT TO
DO TRIALS AND THAT WAS
FASCINATING TO ME BECAUSE IT WAS
PART OF THIS OUTSOURCING THAT I
WAS SEEING BUT ALSO A SCALE
SHIFT THAT WAS PART OF IT. A
LOT OF THE NEW FACILITIES BEING
BUILT WERE REALLY LARGE. SO IF
YOU KIND OF THINK ABOUT MOST
RESEARCH UNITS OF THIS KIND THAT
MIGHT BE AT AN ACADEMIC MEDICAL
CENTER THEY'RE PRETTY SMALL,
PROBABLY 12-16 BED UNITS BUT A
LOT OF THESE FACILITIES WERE
BUILDING 100-200 BED RESEARCH
UNITS. THERE ARE TWO IN THE
U.S. THAT ARE 300 BED UNITS.
THESE ARE ACTUALLY BIGGER THAN
SOME COMMUNITY HOSPITALS. IT
MADE ME WONDER WHO IS
PARTICIPATING IN THESE STUDIES,
HOW ARE THEY GETTING SO MANY
HEALTHY VOLUNTEER TO FILL THESE
FACILITIES SO I CONDUCTED
ETHNOGRAPHIC RESEARCH T
QUALITATIVE RESEARCH.
ON THESE FACILITIES. AND I WENT
TO SIX DIFFERENT FACILITIES IN
THE U.S. AND I REALLY WANTED TO
HAVE A VARIETY OF TYPES OF
RESEARCH SETTINGS SO I WENT TO
ONE ACADEMIC SITE AND FIVE
FOR-PROFIT SITES. ONE OF THESE
WAS A PHARMACEUTICAL SITE. TWO
WERE OWNED AND OPERATED BY
CONTRACT RESEARCH ORGANIZATIONS.
WHICH HAVE KIND OF DIFFERENT
FUNCTION IN THE INDUSTRY AND
THEN TWO WERE JUST KIND OF
INDEPENDENTLY OWNED. THEY
TALKED ABOUT THEMSELVES AS MOM
AND POP RESEARCH OPERATIONS.
AND I ALSO VARIED IT BY WHERE IT
WAS IN THE COUNTRY. I OBSERVED
PROCEDURES AND SAT IN ON
MEETINGS AND I RESEARCHED STAFF
AND VOLUNTEERS. SO THE THING
THAT SURPRISE ME THE MOST WAS
THE DEMOGRAPHICS OF HEALTHY
VOLUNTEERS WHEN I WENT INTO THIS
RESEARCH I KIND OF EXPECT, I
THINK THERE WAS THIS NARRATIVE
OF OH, IT'S YOUNG, WHITE,
COLLEGE STUDENTS THAT ARE
ENROLLING IN THESE STUDIES
BECAUSE THEY NEED SOME BEER
MONEY AND THAT WAS SO FAR FROM
WHAT I FOUND WHEN I ACTUALLY
START DOING THIS RESEARCH THAT
MOST OF THE PARTICIPANTS WERE
MALE AND THAT -- I REFERENCED
EARLIER THAT THE NONHUMAN ANIMAL
STUDIES AREN'T COMPLETE A LOT OF
TIMES WHEN THESE STUDIES BEGIN
THERE ARE A LOT OF RESTRICTIONS
ON WOMEN OF CHILD BEARING
POTENTIAL BUT IT WAS 75-80% MEN
IN THESE FACILITIES AND IT WAS A
DISPROPORTIONATE NUMBER OF
MINORITY PARTICIPANTS SO IN THE
EAST AND THE MIDWEST IT WAS
MOSTLY BLACK OR AFRICAN AMERICAN
PARTICIPANTS AND IN THE
SOUTHWEST IT WAS MOSTLY LATINO
PARTICIPANTS. AND MOST
PARTICIPANTS WERE BETWEEN THE
AGES OF 20 AND 45 BUT IF WE LOOK
AT WELL HOW OLD WERE THEY IN
THAT BRACKET MOST WERE 30S OR
EARLY 40S. THEY WEREN'T AS
YOUNG AS I EXPECTED AND MOST
WERE LOWER INCOME, LOW
EDUCATIONAL ATTAINMENT. AND
THEY WERE PEOPLE WHO HAD AN
UNSTABLE EMPLOYMENT HISTORY AND
I ALSO FOUND THAT MANY ACTUALLY
HAD A HISTORY OF INCARCERATION.
AND MANY WERE IMMIGRANTS. SOME
OF WHOM WERE NOT LEGALLY
PERMITTED TO WORK IN THE U.S. SO
FOR BOTH OF THESE LAST
CATEGORIES WHAT I FOUND IS THIS
BECAME A REALLY IMPORTANT
ECONOMIC OPPORTUNITY TO THOSE
GROUPS WHO MIGHT FACE
DISCRIMINATION. IN THE TYPICAL
JOB MARKET. SO THESE IF A --
FACILITIES DIDN'T ASK IF THEY
HAD A CRIMINAL RECORD OR THEIR
IMMIGRATION STATUS.
FOR ME WHEN WE THINK ABOUT HOW
IMPORTANT IT IS TO DIVERSIFY
MEDICAL RESEARCH AND TO GET
UNDERREPRES
UNDERREPRESENTED GROUPS INTO
RESEARCH. IT MADE ME ASK WHY
DOESN'T THIS HIGHER PERCENTAGE
OF RACIAL AND ETHNIC MINORITIES
FEEL LIKE A SUCCESS? AND SO IN
MY BOOK THAT I PUBLISHED IN 2020
ADVERSE EVENTS, I REALLY TRIED
TO KIND OF UNPACK THIS PARADOX
OF MINORITIES PARTICIPATING IN
THE RISKIEST STUDIES WITH NO
DIRECT BENEFIT TO KIND OF FIGURE
OUT HOW TO ANALYZE THIS, HOW TO
THINK ABOUT THIS FROM AN ETHICAL
PERSPECTIVE AND SO MY ARGUMENT
IN THE BOOK IS THAT RACIAL AND
ECONOMIC INEQUALITIES IN THE
U.S. CREATE A MARKET OF HEALTHY
VOLUNTEERS FOR PHASE ONE TRIALS
SO THAT'S I THINK THE PIECE THAT
MAKES THIS SO UNCOMFORTABLE THAT
REALLY IT IS A SYSTEM THAT IS
EXPLOITING POOR PEOPLE OF COLOR
ESSENTIALLY SO THAT'S WHAT I'M
GOING TO MOSTLY FOCUS ON TODAY
THE BOOK ALSO TALKS ABOUT HOW
THERE ARE VALIDITY CONCERNS THAT
ARE REALLY INHERENT TO HOW THE
PHASE ONE INDUSTRY WORKS AND
THAT ENSURES THAT ANY DRUGS
APPEAR SAFER THAN THEY ARE AND
IF PEOPLE HAVE QUESTIONS ABOUT
THAT I AM MORE TO TALK ABOUT THE
VALIDITY ISSUES AT THE END SO I
WANT TO GIVE YOU A SENSE OF HOW
HEALTHY VOLUNTEERS ACTUALLY TALK
ABOUT WHAT THEY'RE DOING, HOW
THEY'RE THINKING ABOUT WHAT
THEY'RE GOING AND THEY REALLY
FOCUS ON THEIR FINANCIAL
MOTIVATIONS SO, FOR INSTANCE, I
SHOULD SAY ON ALL OF THESE NAMES
ARE PSEUDONYMS LOUIS TOLD ME I
WANTED TO MAKE SOME MONEY.
(READING). AND PEOPLE REALLY
DID FOCUS ON SORT OF THE
DESPERATION THAT DROVE THEM TO
PARTICIPATE. ESPECIALLY THE
FIRST TIME I WOULD SAY. THAT
WAS REALLY PART OF THEIR
NARRATIVE OF WHY THEY KIND OF
DECIDED TO DO SOMETHING LIKE
THIS. AND MANNY'S STORY REALLY
STRUCK ME BECAUSE HE FOUND OUT
ABOUT PARTICIPATING WHEN HE WAS
AT A PAROLE HEARING AND THERE
WERE FLYERS ON THE WALL
ADVERTISING CLINICAL TRIALS SO
HE HADN'T EVEN KNOWN ABOUT IT
BEFORE THAT AND SO HE -- LEA
LEARNING ABOUT IT AND GRANTED
PAROLE HE FELT IT WAS SOMETHING
HE HAD TO DO. HE SAID WHERE
ELSE AM AN I GOING TO GET IT.
MEANING THE MONEY FROM THE
STUDY. CAR'S BROKE DOWN
(READING). MANNY WENT TO A
STATE WHERE HE HAD TO PAY FOR
THE COST OF HIS OWN SUPERVISION.
HE HAD EXTRA FINANCIAL
PRESSURES. ANOTHER EXAMPLE
COMES FROM MIERA SOL WHO SAID
WHAT MADE ME DECIDE TO ENROLL,
TRULY HARDSHIP, THE PEOPLE THAT
GO TO THESE CLINICS OFTEN
THEY'RE PEOPLE THAT BECAUSE OF
LACK OF WORK WE (RWORK WE (READ
EMPHASIZING THIS IDEA THAT IT'S
NOT SOMETHING YOU WANT TO DO
UNLESS YOU HAD TO DO IT AND THIS
WAS DIFFERENT FROM A LOT OF THE
PARTICIPANTS WHO WERE WHITE THAT
I INTERVIEWED. THEY TENDED TO
SEE IT AS A TEMPORARY THING.
SOMETHING THAT THEY IMAGINED
THEY WOULD DO IT ONCE AND THEY
WOULD NEVER HAVE TO DO IT AGAIN
SO FOR INSTANCE, TED SAID I
SUPPOSE IF I DON'T FIND WORK
RIGHT AWAY (READING). SO THIS
BELIEF THAT IT WAS GOING TO BE,
YOU KNOW, SOMETHING THAT WOULD
KIND OF GET THEM OVER THE HUMP,
HELP THEM PAY THEIR RENT OR
MORTGAGE OR SOMETHING LIKE THAT
AND THEN THEY WOULD BE BACK ON
TRACK WHEREAS A LOT OF PEOPLE OF
COLOR DIDN'T HAVE THAT
NARRATIVE. THEY REALLY TALKED
ABOUT HOW CLINICAL TRIALS BECAME
PART OF THEIR STRATEGY TO DEAL
WITH THE PERSISTENT FINANCIAL
PRECARITY THAT THEY WERE
EXPERIENCING SO FOR INSTANCE
WESLEY SAID I THINK I WILL KEEP
DOING THEM UNTIL I GET OLD
(READING). SO DEFINITELY THIS
WORRY OF HOW DIFFICULT IT WAS TO
GET AHEAD. FOR A LOT OF THE
PEOPLE THAT I MET. AT THE SAME
TIME, SO THERE WERE PARTICIPANT
WHO IS WERE DOING IT JUST KIND
OF AS A SURVIVAL STRATEGY BUT
THEN THERE WERE OTHER
PARTICIPANTS THAT SAW YOU COULD
MAKE A LIFESTYLE OUT OF CLINICAL
TRIALS AND THEY TALKED ABOUT
THEMSELVESES NOT AS HUMAN GUINEA
PIGS BUT HUMAN LAB RATS.
BENNETT TALKS ABOUT THIS.
(READING). WHEN BENNETT WAS
TALKING ABOUT MCMCIT WASN'T HYPE
CALENDAR HE HAD ACTUALLY WORKED
AT MCDONALD'S SO HE WAS TALKING
ABOUT THE LIFE HE COULD LEAD IN
THE HOURLY BASED VERSUS CLINICAL
STUDIES. AT THE SAME TIME WE
DID SEE SOME DRAWBACKS HE SAID
THE THING THAT THE LAB RAT THING
LACKS IS CONSISTENCY. IF MY
RENT IS DUE AND THE STUDY GETS
CANCELED MY RENT GETS CANCELED
(READING). AND THEN THE IDEA OF
COMPETITION WAS REALLY A BIG
PART OF THE CLIMATE OR THE
CULTURE OF PARTICIPATION THAT
PARTICIPANTS REALLY DID WORRY
ABOUT BEING ABLE TO GET IN
STUDIES WHEN THEY WANTED IT AND
I WILL COME BACK TO THAT THEME
IN A LITTLE BIT BUT FIRST I JUST
WANTED TO, SO IN A SUBSEQUENT
STUDY THAT MY TEAM AND I
CONDUCTED WE DID LONGITUDINAL
WORK WITH VOLUNTEERS TO TALK
ABOUT THE STUDIES THEY ENROLLED
WITH IN A THREE YEAR PERIOD SO
WE TALKED ABOUT HOW MUCH
CLINICAL TRIALS WERE PAID AND
HOW MUCH PEOPLE WERE EARNING SO
THIS GIVES YOU A SENSE OF HOW
MUCH THE STUDY THE PAID DURING
THAT TIMEFRAME AND YOU CAN SEE
MOST OF THEM PAID UNDER 6,000
DOLLARS BUT THERE WERE SOME THAT
QUITE SUBSTANTIAL IN PAYMENT. I
ALSO JUST WANTED TO GIVE YOU
SOME DATA ABOUT HOW MUCH PEOPLE
EARNED IN A YEAR SO WHAT YOU
COULD SEE IS THAT THE AVERAGE
PARTICIPANT WAS SCREENING FOR
ABOUT THREE STUDIES A YEAR AND
PARTICIPATING IN ONE OR TWO.
AND EARNING ABOUT 4,000 DOLLARS.
SO PEOPLE WERE CERTAINLY NOT
GETTING RICH ON THIS. BUT THIS
IS KIND OF AN AVERAGE ACROSS
PEOPLE. ONE OF THINGS I WANTED
TO POINT OUT TOO. YOU COULD SEE
THERE WAS A THREE YEAR STUDY WE
STARTED WITH 113 PEOPLE IN --
SORRY, 131 PEOPLE IN OUR STUDY
BUT IN THE FIRST YEAR 113 OF
THEM CONTINUED TO SCREEN FOR
TRIALS SO SOME STOPPED
BASICALLY. 14% STOPPED. BUT
YOU CAN SEE THE NUMBER GOES DOWN
EVERY YEAR SO THERE ARE PEOPLE
WHO ARE PARTICIPATING REGULARLY
BUT WE ACTUALLY SAW THAT THE
NUMBER WAS DECREASING AND ONE OF
THE REASONS FOR THIS IS PEOPLE
FELT DISILLUSIONED WITH THE FACT
THAT THEY WEREN'T EARNING AS
MUCH MONEY AS THEY THOUGHT THEY
MIGHT BE ABLE TO. THE NOTE ON
COMPENSATION, PARTICIPANTS
REALLY HAVE CRITICAL STANCES ON
THE INDUSTRY. SO SHIRLEY SAID
THE DRUG COMPANIES, THE ACADEMIC
INSTITUTIONS OR WHATEVER IT IS,
THIS HUGE WHEEL THAT TURNS, YOU
KNOW, TO POWER CLINICAL STUDIES,
(READING). SO THERE'S REAL
FEELING THAT DESPITE HOW WE
THINK THESE REALLY PAY QUITE A
LOT, A SUBSTANTIAL PAYMENT A LOT
OF TIMES THE PARTICIPANTS FELT
IT WAS NOT AS GENEROUS AS IT
MIGHT SEEM. SO I WANT TO GIVE
YOU A LITTLE BIT OF WHAT THE
RISKS THEY FEEL. AND HOW THEY
ACKNOWLEDGE AND RATIONALIZE RISK
AS WELL AS THE RISK OF NOT
QUALIFYING, PARTICIPANTS
DEFINITELY ARE AWARE OF WHAT THE
STUDIES REQUIRE, THEY ARE
READING THE CONSENT FORMS, SOME
OF THESE CLINICS ACTUALLY QUIZ
THEM ON THE CONTENT OF THE
CONSENT FORMS SO THEY KNOW
THERE'S RISK. THIS PARTICIPANT
SAID, (READING). SO THIS
FEELING THAT THERE IS RISK, IT
IS SOMETHING THAT COULD HURT YOU
IN THE LONG-TERM IF YOU ARE NOT
CAREFUL. JULIA SAID I TREAT
EVERY DRUG OR EVERY PROCEDURE
DIFFERENTLY I'M VERY CONCERNED
ABOUT EVERY SIDE EFFECT JUST AS
I WAS THE FIRST TIME BECAUSE
THIS MIGHT BE THE ONE, IT'S A
RISK THAT YOU'RE TAKING, THIS IS
YOUR LIFE AND HEALTH AT STAKE.
AT THE SAME TIME, PARTICIPANTS
REALLY RATIONALIZE THE RISK.
THEY ACKNOWLEDGE THAT THE RISK
WAS OUT THERE BUT THEY ALSO
TENDED TO BELIEVE THEY WEREN'T
GOING TO BE HARMED INDIVIDUALLY
SO THIS PARTICIPATE SAID,
NORMALLY THEY SORT OF TALK ABOUT
HOW LIKE WE TEST THESE DRUGS
(READING). ANOTHER PARTICIPANT
TALKED ABOUT HIS OWN HEALTH AND
YOUTH AND SAID THE BEST TIME TO
DO IT IS WHEN YOU'RE YOUNG AND
YOUR BODY HAS A CHANCE TO REPAIR
ITSELF. WHEN YOU'RE OLD THERE'S
NOT MUCH LEFT IN THE TANK SO I
THINK (READING). AND THIS
ACTUALLY PARALLELS WHAT I WAS
SAYING ABOUT THERE BEING
POTENTIALLY LESS RISK TO HEALTHY
VOLUNTEERS BECAUSE THEY'RE
HEALTHY SO TROY HAS SORT OF
EMBODIED THAT IDEA HERE. A LOT
OF THE PARTICIPANTS, WE TALK
ABOUT TRUST AND DISTRUST OF
RESEARCH BUT A LOT OF THE
PARTICIPANTS WHO ARE IN THESE
STUDIES ACTUALLY HAVE A LOT OF
TRUST IN THE CLINICS AND IN THE
PROCESS. SO AR NELL SAID I'M
TRUST
TRUSTING THE IRB, HE WASN'T SURE
WHAT THAT IS (READING). HOOIB R
HERE HE WAS TALKING ABOUT IF NEW
RISKS EMERGE THEY HAVE TO GIVE
NEW CONCEPT FORMS AND THEY HAVE
A PROCESS OF INFORMING
PARTICIPANTS IN THIS PROCESS
THEY'RE OPERATING ABOVE BOARD,
CAN I TRUST THAT THEY'RE REALLY
TELLING US THE INFORMATION THAT
I NEED TO HAVE. HE SAID I KIND
OF GO IN WITH A SENSE OF
SECURITY THAT EVERYBODY'S DOING
WHAT THEY'RE SUPPOSED TO DO. SO
ONE OF THE THINGS THAT'S
INTERESTING IS WHEN I TALK TO
PARTICIPANTS ABOUT RISK THEY
DIDN'T ANSWER THE WAY I
EXPECTED. THEY WERE NOT TALKING
ABOUT THE RISK OF A CLINICAL
TRIAL THEY WERE TALKING ABOUT
THE RISK OF NOT QUALIFYI IING F
STUDIES. FOR THEM IT WAS THE
RISK TO NOT BE INVITED TO
PARTICIPATE SO I THINK THE SENSE
OF RISK REALLY CAME FROM EITHER
THE COMPETITION THAT WAS OUT
THERE BUT PROBABLY MORE OFTEN
FROM ACTUALLY FAILING A
SCREENING. SO FROM THIS PROCESS
OF SAYING, WELL, I'M A HEALTHY
PERSON, I'M GOING TO GO IN AND
THEN FINDING OUT THAT SOMETHING
WAS WRONG IN THEIR BLOODWORK OR
SOMETHING AND DIDN'T ACTUALLY
MAKE IT INTO STUDIES.
SO THE CONSEQUENCE OF THIS IS
THEY START TO THINK OF STUDIES
DIFFERENTLY AND PICK THEM
CAREFULLY FOR INSTANCE WHEN I
WAS AT ONE OF THE FACILITIES IN
THE NORTHEAST THEY WERE
SCREENING FOR A HEP C TRIAL AND
IT WAS A DOSE ESCALATION STUDY
AND THEY WERE RECRUITING THE
HIGHEST DOSE COHORT FOR THAT
STUDY ARE RECRUITER WAS DOING A
GREAT JOB TALKING ABOUT WHAT TO
EXPECT. PREPARE TO PUKE WAS HER
LINE. AND SO WHAT I STARTED TO
SEE WAS THAT PARTICIPANTS WERE
EITHER DECIDING TO NOT GIVE
THEIR CONSENT AT ALL AND WALK
AWAY OR THEY CONSENTED AND THEY
WENT THROUGH THE SCREENING
PROCESS BUT THEN THE DAY OF THE
STUDY WHEN THEY EXPECTED TWELVE
PARTICIPANTS TO SHOW UP TO CHECK
OUT THE STUDY, ACTUALLY ONLY
FIVE DID AND THE STUDY HAD TO BE
CANCELED IF THEY KNEW IN ADVANCE
THEY WERE GOING TO EXPERIENCE
SIDE EFFECTS. A LOT OF TIMES
PARTICIPANTS ARE ALSO TRYING TO
IMPROVE THEIR CHANCES OF
QUALIFYING FOR STUDIES BY
ENGAGING IN DIFFERENT POST-STUDY
ROUTINES AND SOME OF THESE
THINGS ARE POSITIVE AND SOME ARE
NEGATIVE. EDDIE SAID WHEN WE
GET OUT OF HERE I'M BASICALLY ON
A VITAMIN REGIMEN, SO WE'RE
PUTTING BACK EVERYTHING THEY
TOOK FROM US. HOW MUCH BLOOD
YOU GAVE AND DEPENDING ON HOW
MANY STUDIES YOU DO (READING).
SO I'M A SOCIAL SCIENTIST, I WAS
REALLY STRUCK BY THE LEXICON
THAT THE PARTICIPANTS THAT I WAS
INTERVIEWING WERE USING SO, FOR
INSTANCE, IN THE FIRST FACILITY
THAT I VISITED, THERE WAS A DAY
WHEN ONE OF THE PARTICIPANTS WAS
TELLING ME THAT HE REALLY
STRUGGLED WITH HIS BILIRUBIN AND
I HAD NO IDEA WAS BILIRUBIN WAS.
SO I ASKED HIM ABOUT AND IT HE
TOLD ME ABOUT HOW IT WAS A LIVER
ENZYME AND I REMEMBER GOOGLING
IT THAT NIGHT BUT DEFINITELY
PARTICIPANTS ARE LEARNING ABOUT
THESE THINGS BECAUSE IT MATTERS
TO THEM. THEY'RE WORRIED ABOUT
ENROLLING SO IF THEY FIND OUT
THEY DIDN'T QUALIFY FOR A STUDY
BECAUSE OF THEIR BILIRUBIN NOW
BILIRUBIN IS REALLY IMPORTANT TO
THEM. SO THERE'S ALSO A PROCESS
OF BEHAVIOR CHANGE THAT
PARTICIPANTS ENGAGE IN. SO
THIS, I THOUGHT WAS A GREAT
EXAMPLE THAT CAME UP FROM DON HE
SAID WHEN I FIRST STARTED DOING
STUDIES I WAS SMOKING WEED AND
THAT'S WHAT GOT ME TO STOP
(READING). SO A LOT OF
PARTICIPANTS TALKED ABOUT HOW,
BECAUSE THEY WEREN'T SURE
NECESSARILY WHEN THEY WERE GOING
TO SCREEN FOR A TRIAL -- IF TWO
DAYS LATER THEY WERE GOING FIND
OUT THERE WAS A GREAT STUDY THEY
WANTED TO SCREEN FOR AND MAYBE
THE DRINKING THEY HAD DONE WAS
GOING TO SUDDENLY DISQUALIFY
THEM SO REALLY KIND OF THINKING
ABOUT CLINICAL TRIALS MUCH MORE
SO THAN YOU MIGHT IMAGINE
BECAUSE OF WANTING TO BE ABLE TO
BE COMPETITIVE TO GET IN. SO I
WANT TO SWITCH GEARS A LITTLE
BIT IN THE TIME THAT I HAVE LEFT
BECAUSE, YOU KNOW, REALLY WHAT I
WAS TRYING TO DO WITH THIS FIRST
PART OF THE TALK WAS TO THINK
ABOUT HOW PARTICIPANTS FOCUS ON
THE FINANCIAL COMPENSATION THAT
THEY CAN GET FROM STUDIES SORT
OF DOES LEAD TO THEM EVEN IF
THEY ARE NOT PARTICIPATING AS
PROFESSIONAL PARTICIPANTS IN THE
SENSE OF DOING IT FOR A LIVING
THEY'VE KIND OF BECOME
PROFESSIONAL IN THE SENSE WHERE
THEY BECOME EXPERTS ABOUT THE
PROCESS, THEY'RE THINKING ABOUT
THE PROCESS, THEY MIGHT BE
ENGAGING IN BEHAVIORS FOR THE
PROCESS. SO THIS FINANCIAL
COMPENSATION IS CERTAINLY
MOTIVATING IN A MORE HOLISTIC
WAY THAN THEIR DECISION TO
PARTICIPATE OR NOT. BY I WA
WANT -- BUT I WANTED TO FLIP IT
TOO.
A LOT OF TIMES WE THINK ABOUT
POTENTIAL FOR UNDUE INDUCEMENT
BUT WHAT ABOUT THE FINANCIAL
RISK TO PARTICIPANTS? AND HOW
CAN COMPENSATION FOR RESEARCH
ITSELF ACTUALLY OPPOSE A RISK TO
HEALTHY VOLUNTEERS? SO IF WE
THINK ABOUT COMPENSATION IN THE
CONTEXT OF RESEARCH IN THE U.S.
IT'S TAX INCOME SO HEALTHY
VOLUNTEERS ARE INDEPENDENT
CONTRACTORS FOR TAX PURPOSES
IT'S LIKE BEING A DRIVER FOR
UBERER LYFT. THE GIG ECONOMY.
BUT CONSENT FORMS ONLY PROVIDE
LIMITED INFORMATION ABOUT TAX
LIABILITY THEY SAY SOMETHING
ALONG THE LINES OF, YOU KNOW,
THIS INCOME OR THE COMPENSATION
IS REPORT TO BELIEVE THE IRS.
AND THEN UP TO PARTICIPANTS TO
DETERMINE WHAT DOES THAT MEAN
EXACTLY? AND SO AS PART OF IS
THIS FOLLOW UP STUDY THAT WE DID
TO THE OBSERVATIONAL STUDY I
INITIALLY DID WE STARTED TO
REALIZE THAT PARTICIPANTS WERE
TALKING ABOUT TAXES A LOT OVER
THE THREE YEARS THAT THEY WERE
INVOLVED WITH OUR STUDY. AND
WHAT WE SAW WAS THAT THERE WAS
OFTEN THIS INITIAL BELIEF THAT
IT'S TAX-FREE AND THIS IDEA THAT
BEING TAX-FREE BECAUSE TAXES
AREN'T WITHHELD FROM THEIR
COMPENSATION UNLIKE A PAYCHECK
SO THEY HAD THIS FEELING LIKE
WOW, I'M GETTING THIS LUMP SUM
AND THERE'S NO TAXES. HOWEVER,
FOR PARTICIPANTS WHO,
ESPECIALLY, THOSE WHO DID MANY
STUDIES IN THAT FIRST YEAR THAT
THEY EVER DID STUDIES THEY
REALLY WERE SHOCKED WHEN THEY
RECEIVED A 1099 THE NEXT JANUARY
REALLY HAVING THIS FEELING LIKE
IT WAS OUT OF THE BLUE THAT THEY
DIDN'T EXPECT THAT. SO F IN
KIND OF THINKING ABOUT HOW
PARTICIPANTS TALK TO US ABOUT
THE TAX IMPLICATION OF STUDIES.
THERE WERE TWO WAYS OF THINKING
ABOUT IT. SOME OF THEM
NAVIGATED THE TAX SYSTEM AS AN
ENTREPRENEUR LIKE THEY TALKED
ABOUT HOW IT WAS A JOB SO THEY
NEEDED TO SAVE THEIR RECEIPTS
AND ITEMIZE DEDUCTIONS AND THEY
REALLY HAD A VERY SAVVY STRATEGY
OF TRYING TO THINK ABOUT HOW TO
LIMIT THEIR TAX LIABILITY FROM
STUDIES. BUT MOST PARTICIPANTS
JUST AVOIDED REPORTING THEIR
TRIAL INCOME AND SO THIS CREATED
I THINK A HUGE FINANCIAL AND
SOMETIMES EVEN A LEGAL LIABILITY
FOR PARTICIPANTS. SO I WANTED
TO JUST GIVE SOME OF THIS SOME
SENSE OF THIS AS WELL. SO ROMAN
IN TALKING ABOUT HOW HE USED TO
AVOID REPORTING TRIAL INCOME,
TOLD US, FOR EVERY TIME THAT YOU
TAKE THAT MONEY IT'S TECHNICALLY
(READING). SO HERE HE'S SAYING
IT'S NOT WITHHELD, AND IF YOU
RIDE UNDER THE RADAR (READING).
BUT AT THE SAME TIME AS
PARTICIPANTS BECAME MORE AND
MORE AWARE OF THEIR TAX
LIABILITY THEY STARTED TO
REALIZE THAT TAX DEBT WAS
SOMETHING THAT COULD BE
POTENTIALLY VERY CRUSHING. SO
STEVE WAS AN INDIVIDUAL THAT HAD
STARTED TO PARTICIPATING IN
STUDIES IN THE LATE 1990S SO HE
HAD BEEN DOING STUDIES FOR A
VERY, VERY LONG TIME. AND HE
BELIEVED THAT HE HAD EVENED WELL
OVER 250,000 DOLLARS IN THAT
TIME.
THAT WAS HIS BACK OF THE
ENVELOPE MATH OF FIGURING OUT
HOW MUCH HE HAD PROBABLY GOTTEN
AND IN THAT TIME HE NEVER FILED
HIS TAXES, HE NEVER PAID TAXES.
WHY THIS SUDDENLY BECAME AN
ISSUE WAS THAT HE HAD TRAVELED
OVERSEAS AND MET A WOMAN AND HAD
A CHILD WITH THAT WOMAN AND
WANTED TO BE ABLE TO BRING HER
AND THE CHILD TO THE U.S. AND
SO HE STARTED LOOKING INTO THE
PROCESS OF HOW COULD HE GET HER,
A FIANCE, AS PART OF THIS PLAN
TO BRING HIS CHILD INTO THE U.S.
AND HE REALIZED HE WAS NOT
ELIGIBLE FOR A FIANCE VISA
BECAUSE HE OWED MONEY TO THE IRS
SO THIS WAS A HUGE WAKE-UP CALL
TO HIM AND HE SAID AS HE HAD
BEEN EXPLORING THIS I WILL
PROBABLY OWE ABOUT 35,000
DOLLARS MEANING IN BACK TAXES
AND MOST OF THAT IS WHAT THE IRS
SEES IS ME DOING CLINICAL TRIALS
WHERE THEY DIDN'T TAKE ANY TAXES
OUT I GOT PAID WITH CHECKS
WITHOUT TAXES WITHHELD SO I'M
SUPPOSED TO PAY TAX TO THE IRS
ALL THOSE YEARS FOR ALL THOSE
CLINICAL TRIALS AND EVEN THIS
AMOUNT OF $35,000 WAS, HE WAS
STILL NEGOTIATING WITH THE IRS
BUT IT WAS ALREADY, THEY HAD
ALREADY HALVED IT SO YOU CAN SEE
HOW SIGNIFICANT A TAX BURDEN CAN
BE FOR PARTICIPANTS WHO HAVE
BEEN DOING THIS FOR A REALLY
LONG TIME WE ALSO DID SEE THERE
WERE PERVERSE INCENTIVES
STEMMING FROM TAX LIABILITY.
SOME PARTICIPANTS STARTED TO SAY
THAT THEY COULDN'T PARTICIPANT
IN MORE STUDIES EVEN THOUGH THEY
COULD REALLY USE THE MONEY
BECAUSE THEY WANTED TO STAY
BELOW A CERTAIN INCOME THRESHOLD
SO FOR INSTANCE IF THEY
QUALIFIED FOR GOVERNMENT SUPPORT
THEY DIDN'T WANT TO JEOPARDIZE
THAT SOURCE OF INCOME FOR
THEMSELVES. SOME, THERE WERE A
COUPLE PEOPLE WHO WANTED TO GO
BACK TO COLLEGE AND REALIZED
THAT THEY MIGHT QUALIFY FOR
STUDENT AID BUT THEY WOULDN'T.
IF THEY HAD AN INCOME OVER A
CERTAIN LEVEL SO THEY WERE
REALLY KIND OF WORRIED ABOUT
THESE THRESHOLDS EVEN THOUGH
THEY NEEDED THE INCOME FOR THEIR
DAY-TO-DAY LIVES SO I THOUGHT
THAT WAS REALLY INTERESTING AND
VERY SAD KIND OF THINKING ABOUT
THE KINDS OF DECISIONS THEY WERE
MAKING. PARTICIPANTS ALSO
TALKED ABOUT HOW SOMETIMES THEY
NEEDED TO DO ANOTHER CLINICAL
TRIAL IN ORDER TO PAY THE MONEY
THEY OWED AT THE -- FOR LAST
YEAR'S TAXES SO SOME
PARTICIPANTS SAID SOME PUT IN
YEARS AND (READING). HE WAS A
BIT JOKING ABOUT IT BUT I THINK
THERE WAS DEFINITELY A TRUTH TO
THIS IN THINKING ABOUT SOME
OTHER PARTICIPANTS AND TELLING,
YOU KNOW, WHEN WE INTERVIEWED
THEM ABOUT THEIR STUDY
PARTICIPATION WE OFTEN ASKED
WHAT PROMPTED YOU TO DO THIS
SPECIFIC STUDY AND AT TAX TIME
IT OFTEN WAS SPECIFICALLY ABOUT
TAXES. ANOTHER ISSUE I THINK
ABOUT THIS RISK TO PARTICIPANTS
IS THAT IT MIGHT BE TAKING
ADVANTAGE OF HEALTHY VOLUNTEERS
THAT COMPENSATION IS TAXED
INCOME BECAUSE ACTUALLY IF WE
THINK ABOUT IT, IT'S LESS
COMPENSATION THAN IT SAMES --
SEEMS WHEN MAKING THAT DECISION.
AFTER THE TAXES, THE TIME, THE
THIS, THE THAT, HOW MANY
SCREENINGS -- AT THE END OF THE
DAY IT WAS A LOT LESS THAN YOU
ACTUALLY THOUGHT YOU WERE MAKING
AND THEN PARTICIPANTS I THOUGHT
MADE AN EXCELLENT POINT ABOUT
HOW IT SEEMED UNFAIR TO BE TAXED
ON SOMETHING WHEN YOU'RE TAKING
A PHYSICAL RISK. ESPECIALLY A
PHYSICAL RISK THAT MIGHT BE FOR
THE GREATER GOOD. SO THIS
PARTICIPANT SAID I DON'T THINK
WE SHOULD HAVE TO PAY TAXES ON
THIS MONEY BECAUSE YOU SHOULDN'T
HAVE TO PAY TAXES FOR THIS
BECAUSE WE'RE DOING NOT BUT
SERVICE FOR THE PEOPLE (READ
(READING). SO I'LL CLOSE HERE.
BUT I JUST REALLY WANTED TO
EMPHASIZE THAT THE SYSTEM THAT
WE HAVE IS REALLY COMING OUT OF
THE REGULATORY SYSTEM. BECAUSE
WE HAVE THIS PHASE TRIAL AND
REQUIREMENT FOR DRUGS TO GET
APPROVED BY THE FDA, YOU KNOW,
WE REALLY HAVE THIS NEED FOR
HEALTHY VOLUNTEERS IN ORDER TO
HAVE DRUGS BE APPROVED. AND SO
WE COULD KIND OF THINK ABOUT
THAT AS CREATING AN INDUSTRY FOR
HEALTHY VOLUNTEER TRIALS AND
THOSE WHO ARE INCENTIVIZED BY
FINANCIAL COMPENSATION BUT ALSO
HAVE THE TIME AND ABILITY TO BE
CONFINED FOR LONG PERIODS OF
TIME AND SO THE U.S. CONTEXT OF
SOCIAL AND ECONOMIC INEQUALITIES
FUNNEL LOW INCOME PEOPLE OF
COLOR INTO THESE TRIALS BECAUSE
THEY'RE OFTEN THE PEOPLE WHO
DON'T HAVE BETTER OPTIONS TO
EARN INCOME. AND I THINK, YOU
KNOW, IF WE ALSO REFLECT ON THIS
COMPENSATION ISSUE I THINK
THERE'S SO MUCH MORE TO THINK
ABOUT BEYOND THIS WORRY ABOUT
UNDUE INDUCEMENT BECAUSE THERE
ARE THESE ETHICAL ISSUES THAT
ARE TIED TO THE PROMISE OF
FINANCIAL FREEDOM THAT
PARTICIPANTS MIGHT HAVE BUT ALSO
THAT COMPENSATION MAY APPEAR
SUBSTANTIAL BUT DOESN'T PROVIDE
THE FINANCIAL SECURITY AND EVEN
COMES WITH FINANCIAL AND LEGAL
RISKS FROM TAXES SO I WILL END
HERE BUT I AM VERY HAPPY TO TAKE
QUESTIONS OR COMMENTS.
>> AUDIENCE: (APPLAUSE).
>> THANK YOU VERY MUCH AS I
MENTIONED FOLKS IN HERE ARE
WELCOME TO GO TO THE MICS AND
ASK QUESTIONS. LOOKS LIKE BOTH
BRITTANY AND DANIEL ARE HEADED
THERE AND I WILL MONITOR FOR
QUESTIONS FROM OUR VIDEO
AUDIENCE. BRITTANY, GO AHEAD.
>> AUDIENCE: I'M WONDERING ABOUT
HOW PARTICIPANTS HEAR ABOUT
STUDIES, YOU MENTIONED THE
PERSON WHO HEARD ABOUT IT ON A
FLYER OUTSIDE HIS PAROLE HEARING
BUT IS IT LARGELY WORD OF MOUTH
IS THERE KIND OF TARGETED GO TO
THIS NEIGHBORHOOD AND POST
FLYERS OR HOW ELSE DO THEY HEAR
ABOUT IT?
>> JILL: IN LARGE CITIES IT'S
ADVERTISED IN PUBLIC
TRANSPORTATION ROUTES. THAT'S
ONE WAY. IN ASKING PEOPLE HOW
THEY LEARNED ABOUT CLINICAL
TRIALS INITIALLY ALMOST EVERYONE
IT WAS WORD OF MOUTH SO I THINK
IT WAS OFTENTIMES THIS THING
WHERE, IT'S -- I THINK IT'S NOT
THAT PEOPLE HAVE NEVER HEARD OF
IT BEFORE THAT BUT I THINK
HAVING MEETING SOMEBODY WHO HAS
DONE IT BEFORE OFTEN GIVES THEM
A SENSE OF SAFETY THAT,' WELL
MY FRIEND OR BROTHER DID IT AND
THEY GOT THROUGH IT OKAY.
AND THAT'S REASSURING THAT IF
THEY'RE OKAY I WILL BE TOO AND
THE MONEY SOUNDS GREAT.
>> AUDIENCE: I WAS ALSO
WONDERING ABOUT HOW MANY
PARTICIPANTS HAD CHILDREN
BECAUSE IT SEEMS LIKE THAT MIGHT
BE A BARRIER TO THE PRIMARY
CAREGIVER PARTICIPATING IN
STUDYING AND IF THEY HAD
CHILDREN WHO WAS THE CAREGIVER
FOR THEM WHILE THEY WERE
INPATIENT.
>> JILL: I WOULD SAY MOST OF
THEM DID OR DO HAVE CHILDREN.
AND THAT -- THAT RAISES A WHOLE
BUNCH OF ISSUES BECAUSE OF
COURSE LOTS OF PEOPLE WERE VERY
CONCERNED ABOUT WHAT WAS
HAPPENING ON THE OUTSIDE SO, YOU
KNOW, FIRST THING THAT CAME TO
MIND WAS A STORY OF A
PARTICIPANT WHO HIS SON GOT
SECOND DEGREE BURNS WHILE HE WAS
IN A STUDY BECAUSE HE PULLED A
LOT POT OFF THE STUFF AND
PARTICIPANT ASKED THE RESEARCH
CLINIC IF HE COULD LEAVE TO MEET
HIS PARTNER AT THE EMERGENCY
ROOM AND THEY SAID YOU'RE
WELCOME TO WITHDRAW FROM THE
STUDY BUT WE CAN'T LET YOU LEAVE
JUST TO GO SO HE WAS VERY TORN
ABOUT THAT DECISION. HE
ULTIMATELY DECIDED TO STAY IN
THE STUDY BUT ALSO FELT VERY
MUCH LIKE A BAD FATHER THAT HE
MADE THAT CHOICE. BUT I THINK,
YOU KNOW, TO ANSWER YOUR
QUESTION ABOUT CAREGIVING, I
THINK, YOU KNOW, IT TIP --
BECAUSE MOST OF THESE
PARTICIPANTS WERE MALE, WERE
MEN, IT TYPICALLY FELL ON THEIR
FEMALE PARTNERS SOMETIMES,
MOTHERS OR MOTHERS-IN-LAW WERE
TAKING CARE OF CHILDREN
INTERESTINGLY A LOT OF THE WOMEN
WITH CHILDREN TALKED ABOUT BEING
THE IN THE STUDIES AS A
VACATION.
BECAUSE THEY HAD AN OPPORTUNITY
TO BE AWAY FROM THEIR KIDS FOR A
FEW DAYS OR FOR A WEEK AND VERY
FEW MEN TALKED ABOUT IT AS A
VACATION BUT A LOT OF WOMEN DID.
>> AUDIENCE: THANK YOU.
>> AUDIENCE: THIS WAS SO
FASCINATING, THANKS SO MUCH FOR
THIS TALK. MY QUESTION WAS, SO
PRESUMABLY A LOT OF THESE
PARTICIPANTS OR MAYBE LIKE --
ARE PARTICIPATING IN STUDIES
DONE BY PRIVATE PHARMACEUTICAL
COMPANIES AND PERHAPS ALSO,
LIKE, GOVERNMENT FUNDED
RESEARCH. LIKE STUFF WITH THE
NIH, I'M WONDERING IF THERE WAS
ANY EVER, LIKE, IF THEY EVER HAD
ANY COMPARISONS BETWEEN THOSE
TWO, YOU KNOW, I GUESS MY
BROADER QUESTION IS
PRIVATIZATION OF THESE PHASE ONE
TRIALS HAD ANY EFFECT ON
SUBJECTIVE EXPERIENCES OR THE
MARKET IN GENERAL?
>> JILL: THAT'S A GREAT QUESTION
AS A ROLE PARTICIPANTS SAW THE
PRIVATE TRIALS AS THE BEST ONES
BECAUSE THEY PAID THE MOST. SO
I THINK A LOT OF TIMES THEY SAW
ACADEMIC MEDICAL CENTERS AS THE
ONES THAT HAD THE REALLY CHEAP
STUDIES THE WAY THEY DESCRIBE
THEM AND THE STUDIES THAT WERE
HAPPENING AT PLACES LIKE NIH,
THEY SAW A AS MOSTLY VACCINE
RELATED.
THOSE WERE NOT NECESSARILY THE
RISKS THEY WERE WORRIED ABOUT
BUT HOW LONG THEY WOULD BE OUT
OF THE STUDY POOL IF THEY DIDN'T
HAVE TO STAY AT THE FACILITY BUT
THEY HAD TO BE FOLLOWED FOR A
YEAR OR SOMETHING LIKE THAT. SO
THEY OFTEN REALLY DID NOT LIKE
THOSE KINDS OF STUDIES. AND A
LOT OF THE PRIVATE FACILITIES,
NOT ONLY DID THEY PERCEIVE THEM
AS PAYING MORE BUT AS MORE
CATERING TO PARTICIPANTS. A LOT
OF THE ACADEMIC MEDICAL CENTERS
THEY KIND OF DESCRIBED AS NOT
HAVING VERY MANY FACILITIES OR
AMENITIES FOR PARTICIPANTS
WHEREAS THE CLINICS HAVE
RECREATION AREAS, TVS, VIDEO
GAMES, ALL KINDS OF STUFF FOR
THEM TO DO. SO I THINK THEY SAW
THEM AS A BETTER ENVIRONMENT
WHEN THEY KNEW THEY HAD TO BE
THERE POTENTIALLY FOR WEEKS SO
THEY WERE COMPARING THE TYPES OF
STUDIES, THE MONEY THAT THEY
WERE PAYING BUT ALSO THE
FACILITIES WHEN THEY WERE KIND
OF THINKING ABOUT WHERE THEY
WANTED TO DO STUDIES, YEAH.
SN.
>> AUDIENCE: HI, THANK YOU FOR
YOUR PRESENTATION, WHEN YOU
TALKED ABOUT PEOPLE TAKING CARE
OF THEIR HEALTH IT REMINDED ME
OF ATHLETES AND HOW THEY HAVE TO
MANAGE THAT STUFF. YOU
MENTIONED TOWARDS THE BEGINNING
BEGINNING THAT THERE WERE
CONCERNS ABOUT VALIDITY AND, YOU
KNOW, MAYBE IT'S THE CASE THAT
SOME OF THESE DRUGS ARE NOT AS
SAFE IT HAS THEY'RE MADE OUT TO
SEEM. SO I WAS WONDERING IF YOU
COULD SAY A LITTLE BIT MORE
ABOUT THAT.
>> JILL: YES I'M HAPPY TO.
ANOTHER PART OF THE STORY ABOUT
HOW PARTICIPANTS ENGAGE IN THESE
BEHAVIORS IS THAT THEY ALSO
BREAK A LOT OF RULES BY ENGAGING
IN THESE BEHAVIORS. ONE OF THE
MAIN THINGS THAT IS REQUIRED OF
PARTICIPANTS IS THEY OBSERVE A
WASHOUT PERIOD BETWEEN STUDIES
AND THAT'S USUALLY 30 DAYS. BUT
IF PARTICIPANTS WANT TO MAXIMIZE
THE INCOME THEY CAN EARN THEY
OFTEN DON'T WANT TO OBSERVE THE
30 DAY WASHOUT PERIOD SO THAT'S
ONE WAY THAT THERE'S SOME
CONCERN ABOUT WHAT, YOU KNOW,
WHAT DOES THAT MEAN FOR THE
VALIDITY OF STUDIES IF THEY'RE
ENROLLING MUCH MORE QUICKLY THAN
THEY SHOULD. SOME OF THEM ARE
TAKING VITAMINS AND SUPPLEMENTS
AND THEY SMUGGLE THEM INTO
FACILITIES. AND THE BIGGEST
CONCERN IS WHETHER THEY'RE
REPORTING ANY ADVERSE EVENTS
THEY EXPERIENCE. OF COURSE
THERE'S THE OBJECTIVE MEASURES
THAT ARE HAPPENING LIKE ALL THE
BLOOD DRAWS OR E KRSECGS AND TH
LIKE THAT BUT ANOTHER BIG PART
OF USING HUMANS IS GETTING THEIR
SUBJECTIVE REPORTS. YOU
WOULDN'T KNOW SOME THINGS UNLESS
THEY TOLD YOU FOR INSTANCE.
PARTICIPANTS AREN'T SURE,
NECESSARILY, WHEN OR IF THEY
SHOULD REPORT SO SOMETIMES
THAT'S BECAUSE THEY'RE WORRIED
THAT REPORTING MIGHT EFFECT
THEIR FINANCIAL COMPENSATION,
SOMETIMES THEY'RE WORRIED
THEY'RE GOING TO GET KICKED OUT
OF A STUDY IF THEY REPORT.
SOMETIMES THEY JUST DECIDE, WELL
I DON'T THINK THIS WAS DRUG
RELATED. I THINK IT'S JUST
BECAUSE I HAVEN'T HAD COFFEE IN
TWO DAYS AND THAT'S A LEGITIMATE
THING. ANY OF US WHO HAVE HAD
CAFFEINE WITHDRAWAL HEADACHE
THAT MIGHT RESONATE WITH US. SO
THEY'RE NOT NECESSARILY DOING IT
FOR THE WRONG REASONS BUT THEY
STILL DO NOT REPORT SOMETHING
THEY'VE EXPERIENCED. SO THAT'S
THE HEALTHY VOLUNTEER BEHAVIOR
BUT ON THE STAFF SIDE I ALSO SAW
A LOT OF RULE BREAKING AS WELL.
SO SOMETIMES STAFF ALLOWING
PARTICIPANTS TO REPEAT LABS BY
LIKE COACHING THEM ABOUT WHAT TO
DO SO THEY COULD GET INTO A
STUDY. SOMETIMES RECORDING THE
WRONG WEIGHT FOR A PARTICIPANT
IF THEY WERE GOING TO, LIKE,
EDGE OUT A LITTLE BIT WITH THEIR
BMI. THE OTHER THING IS THAT
GOING BACK TO THAT WASHOUT
PERIOD, MOST OF THE TIME I WOULD
SAY THE STAFF KNEW IF SOMEBODY
HADN'T OBSERVED THEIR WASHOUT
PERIOD OR SUSPECTED THEY HADN'T
BECAUSE THEY WOULD COME IN WITH
FRESH PUNCTURE MARKS ON THEIR
ARMS OR HAVE ADHESIVE MARKS AND
THE STAFF WOULD SAY I ASKED THEM
IF THEY PARTICIPATED AND THEY
SAID THEY DIDN'T SO I CAN TAKE
THEM BY THEIR WORD SO I FEEL
THERE'S THIS COMPLICITY IN THE
INDUSTRY THAT EVERYBODY KIND OF
WANTS THE HEALTHY VOLUNTEERS TO
BE IN THERE. THEY WANT TO BE IN
THERE, THE STAFF WANT THEM TO BE
IN THERE AND SO I THINK IT DOES
REALLY RAISE CONCERNS ABOUT,
LIKE, HOW DO WE REALLY THINK
ABOUT THE DATA THAT ARE BEING
PRODUCED IF THERE ARE THESE
INCENTIVE TO GAME THE SYSTEM IN
THESE WAYS.
>> YOU MENTIONED THE STAFF
PREFERS TO HAVE THESE
REPEAT "REPEATERS" IN PART
BECAUSE THEY'RE FAMILIAR AND
SORT OF KNOW THE DRILL.
>> JILL: EVEN ON THAT POINT
STAFF AS WELL AS PARTICIPANTS
TOLD ME THAT THEY WOULD BAN OR
PUT ON A BLACKLIST PARTICIPANTS
WHO WERE SEEN AS THOSE WHO
REPORTED A LOT OF ADVERSE
EVENTS.
>> INTERESTING.
>> JILL: THE STAFF JUSTIFIED IT
BY SAYING THESE ARE PEOPLE WHO
ARE JUST TOO SENSITIVE WHICH YOU
KNOW THAT'S PROBLEMATIC BECAUSE
THE PATIENT POPULATION IS GOING
TO VARY BUT I KIND OF ALSO SAW
IT AS THEY CREATE A LOT MORE
WORK FOR THE STAFF IF THEY'RE
DOING A LOT OF AE REPORTING AND
PAPERWORK AS A RESULT SO THAT IS
ANOTHER BIG ASPECT OF IT SO THE
REPEAT PARTICIPANT WASN'T JUST
FOR PEOPLE'S COMPLIANCE OR
ADHERENCE TO THE RILES --
RULES, IT WAS HOW MUCH ARE THEY
A REPORTER OR NOT REPORTED AES.
>> AUDIENCE: HI, THANK YOU, I
HAVE A LOT OF QUESTIONS BECAUSE
THIS IS VERY INTERESTING BUT I
WILL KEEP IT TO TWO FOR NOW.
THERE WAS A SLIDE WHO MENTIONED
SOMEBODY TALKING TO OTHER
PARTICIPANTS ABOUT WHAT VITAMIN
TO TAKE AND YOU WERE TALKING
ABOUT REPEAT PARTICIPANTS.
AND FAMILIARITY WITH STAFF. I'M
CURIOUS IF YOU OBSERVED
FAMILIARITY BETWEEN THE
PARTICIPANTS. AND WHAT THAT
LOOKED LIKE.
>> JILL: YES, THERE ARE FOLKS
WHO TRAVELLED TOGETHER AND
SOMETIMES WHEN THEY DO THESE
STUDIES THEY HAVE TO GO TO
CLINICS THAT ARE NOT THAT CLOSE
TO EACH OTHER SO DOING ROAD
TRIPS TOGETHER. ALTHOUGH THAT
CREATES PROBLEMS IF ONE GETS
INTO A STUDY AND OTHERS DON'T
BUT, YEAH, THEY DEFINITELY HAVE
THESE COMMUNITIES. SOME PEOPLE
HAVE GOTTEN INTO RELATIONSHIPS,
AT LEAST ONE PERSON MARRIED
SOMEONE THAT THEY HAD MET IN A
STUDY. SO THEY DEFINITELY MAKE
FRIENDSHIPS. I WOULD SAY THAT
ONE THING THAT HAPPENS WITH,
BECAUSE THEY'RE REPEAT
PARTICIPANTS IS THEY DON'T
NECESSARILY DECIDE IN ADVANCE TO
DO STUDIES TOGETHER BUT ONCE
THEY GET THERE THEY REALIZE I
KNOW THESE PEOPLE AND THAT
REALLY DOES FOR THE MOST PART
PROBABLY THERE ARE SOME NEGATIVE
EXAMPLES TOO BUT FOR THE MOST
PART ADDS TO THE ENJOYMENT OF
THE STUDY BECAUSE IT IS THEM
SEEING THEIR OLD FRIENDS AGAIN
THEY'RE MOTIVATED BY THE
FINANCIAL COMPENSATION BUT ALSO
MOTIVATED BY THE PEOPLE THEY
WORK WITH.
>> AUDIENCE: YEAH, THAT'S VERY
INTERESTING, THANK YOU AND I WAS
ALSO WONDERING, SO A LOT OF
THESE PLACES YOU'RE STAYING IN A
FACILITY FOR SEVERAL DAYS DID
YOU TALK TO ANY PEOPLE THAT HAD
UNSTABLE HOUSING CONDITIONS THAT
DID IT FOR THAT REASON OR, LIKE,
WHAT WAS THE SITUATION THERE?
>> JILL: YEAH, DEFINITELY THERE
WERE SOME FOLKS WHO DID HAVE
UNSTABLE HOUSING SITUATIONS.
VERY FEW PEOPLE WOULD BE WHAT WE
WOULD THINK OF AS UNHOUSED.
THERE WERE PEOPLE LIVING OUT OF
THEIR CARS BECAUSE THEY TALKED
HOW IT WAS GREAT TO BE IN A
FACILITY WITH A BED, HOT SHOWER
AND MEALS. THEY PROBABLY
WEREN'T THE NORM I THINK THEY
WERE MORE THE EXCEPTION. I
THINK MOST PEOPLE DID TYPICALLY
HAVE A HOME OR LIVING WITH
RELATIVES OR THINGS LIKE THAT
BUT ABSOLUTELY. I THINK THERE
WAS A SENSE OF VULNERABILITY FOR
A LOT OF THESE PEOPLE WHO WERE
DOING STUDIES. BUT I THINK SOME
WERE HOPING THAT THIS WAS GOING
TO KIND OF BE THE THING THAT GOT
THEM AHEAD SO THEY COULD GET
THEIR OWN APARTMENT OR THINGS
LIKE THAT BUT I WOULD SAY MOST
WEREN'T PEOPLE WHO WERE INHOUSED
IN THAT KIND OF CLASSIC SENSE.
YEAH.
>> AUDIENCE: ARE THERE ONLINE
QUESTIONS.
>> YOU CAN TAKE THE LAST TWO
MINUTES IF YOU LIKE.
>> AUDIENCE: OKAY, THERE'S
CLEARLY LIKE A LOT OF TENSION
HERE BETWEEN, LIKE, I DON'T
KNOW, HAVING COERCIVE AMOUNTS OF
MONEY AND THEN ALSO, LIKE,
PEOPLE WHO ARE DESPERATE, LIKE,
TAKING ANY AMOUNT OF MONEY AND
FEELING LIKE THAT'S O KOER
SIEVE. I'M CURIOUS HOW YOU
THINK THAT COULD BE RESOLVED.
>> JILL: WHEN WE WORK WITH
PARTICIPATION AND COMPENSATION I
FEEL THAT FRAMEWORKS BETTER THAN
HEALTHY TRIALS. THEN THE
FINANCIAL COMPENSATION HAVING
THIS OUTSIZED ROLE IN PEOPLE'S
DECISION-MAKING.
WHEN THEY MIGHT ALSO HAVE
MEDICAL BENEFITS. FOR INSTANCE.
BUT I FEEL LIKE WITH HEALTHY
VOLUNTEERS THEY'RE ALREADY DOING
IT FOR THE MONEY. SO IF WE TAKE
THAT AS THE STARTING POINT,
WE'RE, YOU KNOW, INDUCEMENT IS
ALREADY KIND OF THE BASIS OF ALL
OF THIS. SO WHAT BECOMES UNDUE?
THAT I THINK IS REALLY
PROBLEMATIC BECAUSE IF WE LOWER
COMPENSATION TOO MUCH, THEN
YOU'RE GOING TO HAVE
THE "DESPERATE" PEOPLE WHO ARE
THE MOST LIKELY TO DO IT BECAUSE
THEY WILL SEE THAT'S NOT WORTH
MY TIME. I'M MUCH MORE
CONCERNED ABOUT EXPLOITATION OF
PEOPLE WHO ARE IN THESE
PRECARIOUS FINANCIAL SITUATIONS
THAN I AM ABOUT UNDUE INDUCEMENT
BECAUSE THEY ARE DOING IT FOR
THE MONEY. AND EVEN IF WE THINK
ABOUT, YOU KNOW, WHAT HEALTHY
VOLUNTEERS MIGHT SAY ABOUT THIS,
YOU KNOW, THEY'RE COUNTING ON
THE FACT THAT RISKS ARE ALREADY
MINIMIZE. SO, YOU KNOW, FOR
THEM THEY'RE KIND OF THINKING,
WELL, IF THE RISK DOESN'T
CHANGE, WHAT'S THE DIFFERENCE
BETWEEN PAYING ME 2,000 DOLLARS
VERSUS PAYING ME 6,000 DOLLARS
SO PAY ME THE 6,000 DOLLARS.
SO I THINK THAT'S ANOTHER REALLY
BIG PIECE OF IT, YEAH.
>> AUDIENCE: GREAT, THANK YOU SO
MUCH.
>> THANK YOU VERY MUCH. SO NOW
WE'RE BACK TO ME. AND WE'RE
GOING TO DO A CASE AND I'M
HOPING THAT SLIDO IS GOING TO BE
OUR FRIEND TODAY. SO FIRST, MY
GENERAL DISCLAIMER, YOU'VE SEEN
MANY TIMES, THE NEXT IS THAT
WE'RE GOING TO USE SLIDO.COM
AGAIN AND THIS IS THE NUMBER
THAT I'M GOING TO HAVE YOU ENTER
IN ORDER TO ANSWER THE QUESTIONS
I'M GOING TO ASK YOU. AND I'M
GOING TO SHOW THIS NUMBER AGAIN
BUT JUST, I'LL JUST READ IT OUT
FOR FOLKS, IT'S 2918976. SO LET
ME MOVE FORWARD WITH OUR CASE
SO, IMAGINE THAT, SO, THIS IS A
POST-COVID CLINICAL TRIAL FOR
THOSE WHO ARE EXPERIENCING A
POST-COVID SYNDROME. (READING).
SO THESE ARE FOLKS WHO HAD COVID
BAD ENOUGH TO BE HOSPITALIZED,
ARE LEAVING THE HOSPITAL NOW,
GOING BACK HOME BUT HAVE
LINGERING PULMONARY SYMPTOMS.
THE PATIENTS WILL BE RANDOMIZED
TO RECEIVE BB 8 OR A PLACEBO FOR
TWELVE WEEKS WITH A ONE YEAR
FOLLOW UP. ALL PATIENTS WILL
RECEIVE THE SAME BACKGROUND
CURRENT STANDARD OF CARE.
RIGHT? SO JUST TO REMIND YOU
ALL THE IDEA HERE IS THAT
THEY'RE COMING INTO THE HOSPITAL
FOR THIS CLINICAL TRIAL, BUT
THEY ARE EXPECTED TO HAVE
SOMEONE OUTSIDE WHO IS TAKING
CARE OF THEIR PRIMARY CARE BUT
IF ISSUES COME UP DURING THE
PROCESS OF THE STUDY, THAT IS
STANDARD OF CARE, THE HOSPITAL
WILL PROVIDE OR COVER THE COST
OF THAT. SO THE TRIAL IS A
COLLABORATION BETWEEN THE NIH
AND A PRIVATE COMPANY AS THE
COVID PANDEMIC HAS SLOWED THE
COMPANY IS WORRIED ABOUT MEETING
THEIR ENROLLMENT GOALS SO THE
TRIAL IS CURRENTLY UNDERWAY IN
FOUR -- IN SITES IN FOUR STATES.
ONE OF THESE SITES IS IN STATE
X. AND THEY HAVE INDICATED
(READING). MEANING THEY HAVE NO
HEALTH INSURANCE COVERAGE. THE
HEALTH SYSTEM OF WHICH THEY ARE
(READING). SO, HERE IS YOUR
FIRST QUESTION. IS IT OKAY FOR
THE STATE X SITE TO LIMIT
ENROLLMENT IN THIS WAY? AND SO
THE FIRST QUESTION IS JUST A YES
OR NO. DO YOU THINK IT'S OKAY
AND WE'LL OF COURSE TALK ABOUT,
YOU KNOW, FURTHER ABOUT SOME OF
THE ISSUES BUT JUST YOUR GUT
RESPONSE. IS IT OKAY FOR THE
STATE TO LIMIT ENROLLMENT IN
THIS WAY? THANK YOU, ALL, FOR
PARTICIPATING, IT LOOKS ABOUT A
THIRD OF YOU SAY IT'S OKAY AND
ANOTHER TWO-THIRDS SAY IT'S NOT.
SO, OOPS, THAT'S -- INTERESTING
I I THOUGHT I HAD, I'M ASKING
YOU THE QUESTION NEXT. SO OF
THOSE OF YOU WHO SAID YES OR CAN
THINK OF A REASON WHY, WHY DID
YOU SAY YES? OR IF YOU SAID NO,
WHY DO YOU THINK SOMEONE WHO
SAID YES MIGHT SAY YES? AND
THESE ARE OPEN TEXT, YOU CAN
JUST CHIME IN WITH YOUR RESULTS.
GREAT SO ONE WOULD BE TO PREVENT
TOO MANY COSTS TO PARTICIPANTS.
RIGHT? IF THE SITE IS NOT GOING
TO COVER IT, THAT MEANS THAT THE
PARTICIPANT IS GOING TO HAVE TO
COVER THAT EXPENSE. GREAT. SO
SOMEONE SAID, I WAS TAKING THIS
FROM A VERY NARROW PERSPECTIVE
THE QUESTION OF IS IT LEGAL TO
DO THIS, YES, I THINK WE COULD
DECIDE THAT MAYBE IT'S LEGAL TO
DO THAT. BUT WE'RE GOING TO BE
TALKING ABOUT WHETHER IT'S
ETHICALLY APPROPRIATE. LET'S
SEE, IF IT'S DONE DURING THE
PROTOCOL DEVELOPMENT AND IS
AGREED UPON BY ALL THE SITES
THEN MAYBE THAT'S OKAY. THEY
MIGHT NOT BE ABLE TO GET THEIR
FOLLOW UP CARE NEED. RIGHT.
THAT'S A PARTICIPANT FOCUSED
CONCERN. AND BECAUSE IT'S STILL
IMPROVED SOME ACCESS TO
PARTICIPANTS. GREAT. EVERYONE
SHOULD RECEIVE EQUAL TREATMENT
OR OPPORTUNITY. SO I GUESS WE
CAN TAKE THAT IN TWO DIFFERENT
WAYS, ONE, YOU CAN SAY THAT THAT
MEANS THAT IT WOULD -- IT'S OKAY
FOR THEM TO DO THIS. IF THEY
SPREAD THE OPPORTUNITY WIDELY
AMONG THOSE WHO REMAIN. OR SORT
OF THE OPPOSITE, IT PERHAPS
UNFAIRLY EXCLUDES A NUMBER OF
PEOPLE. AS LONG AS IT'S SET UP
AS AN EXCLUSIONARY CRITERIA
UPFRONT IT'S OKAY. THANK YOU
FOR ALL OF THAT. NOW WE'RE
GOING TO MOVE ONTO THOSE WHO
SAID NO OR FOR THOSE WHO SAID
YES BUT IMAGINE WHAT SOMEONE WHO
SAID NO WOULD SAY AND WE STARTED
TO GET SOME OF THOSE IN THE
RESPONSE TO THE LAST ONE. OH,
ACTUALLY, NO, I ALREADY STARTED
THIS, WOW, SO I ASKED THE NEXT
QUESTION WAS TO PUT IT INTO
ETHICS TERMS WHICH MANY OF YOU
HAVE ALREADY DONE. AND SO I
JUST WANT TO HIGHLIGHT A COUPLE
OF THOSE. YOU KNOW, FOLKS
TALKED ABOUT EQUAL OPPORTUNITY.
AND THINKING ABOUT WEATHER IT'S
-- WHETHER IT'S FAIR.
IS IT FAIR TO DO IT OR NOT? LET
ME JUST LOOK. PROMOTING HEALTH
AND SAFETY OF THE SUBJECTS.
RIGHT, BY EXCLUDING THEM, WE ARE
PROTECTING THEM. AND SO NOW I
AM GOING TO GO ONTO THE QUESTION
OF THOSE WHO SAID NO. OTHER
REASONS. TO SAY NO. SAY THAT
IN A SLIGHTLY DIFFERENT WAY.
WHY SHOULDN'T STATE X LIMIT UP
-- ENROLLMENT IN THIS WAY?
UNFAIR EXCLUSION OF THE
UNINSURED. PERHAPS INEQUITABLE.
IT'S MAYBE SEEN AS
DISCRIMINATORY AGAINST THOSE WHO
ARE UNINSURED. YEAH.
EXCLUDEING THEM IS NOT
EQUITABLE. THE PROTOCOL SHOULD
ACCOUNT FOR THIS AND MAKE SURE
THE PARTICIPANTS RECEIVE THE
SAME CARE THAT OTHERS ARE
RECEIVING. WOULD LIMIT
SCIENTIFIC VALIDITY. YES, THANK
YOU. UNFAIR EXCLUSION WITHOUT
INSURANCE COULD BE A LARGE
DEMOGRAPHIC AND MAY HAVE OTHER
CHARACTERISTICS BY EXCLUDING
THEM YOU MAY NOT THEN BE ABLE TO
GENERALIZE TO THE LARGER
POPULATION. GREAT. LOTS OF
GOOD WORDS HERE. JUSTICE,
UNFAIR EXCLUSION, LIMITING
SCIENTIFIC VALIDITY. LESS
REPRESENTATIVE. YEAH. SO
IT -- LOTS OF GOOD TERMS HERE.
COULD IMPACT GENERALIZABILITY.
DISCRIMINATES AGAINST THOSE
WITHOUT INSURANCE. GREAT. AND
SO YOU'VE ALREADY, I'M REALLY
EXCITED, WEEK SIX, YOU'RE
ALREADY, I DON'T EVEN -- I DON'T
HAVE TO ASK YOU TO PUT THESE
INTO THE TERMS OF SOMEONE, OF
THINKING ABOUT ETHICS, YOU ARE
ALREADY PUTTING THEM INTO ETHICS
TERMS WHICH IS AWESOME. OKAY.
SO WHAT I WANT TO DO NOW IS JUST
COVER A COUPLE OF -- RECOVER A
COUPLE OF THE ISSUES THAT WE
ALREADY TALKED ABOUT AND THEN
ASK ONE MORE QUESTION. SO
YOU'VE ALREADY -- WE'VE ALREADY
TALKED ABOUT SOME OF THESE. SO
PARTICIPANTS SHOULD BE CHOSEN
BIASED PRIMARILY ON THE
SCIENTIFIC OBJECTIVES OF THE
TRIAL SO ISSUES AROUND
SCIENTIFIC VALIDITY, RIGHT? IF
SOMEONE IS MEDICALLY ELIGIBLE
ACCORDING TO THE CRITERIA, ONE
COULD SAY THAT EXCLUDING A
PARTICULAR GROUP BASED ON WHAT
WE MIGHT CALL A MORALLY
IRRELEVANT CHARACTERISTIC WOULD
BE INAPPROPRIATE. PARTICIPANTS
SHOULD BE CHOSEN IN A WAY THAT
REDUCES RISK AND ENHANCES
BENEFITS BOTH TO INDIVIDUALS AND
SOCIETY. RIGHT? SO, AGAIN, IF
THE GOAL HERE IS TO THINK ABOUT
BOTH THOSE WHO MIGHT BE
ELIGIBLE, THOSE WHO BENEFIT IF
ENROLLED AND THOSE PATIENTS IN
THE LONG RUN WHO MAY BENEFIT
FROM THE CONTRIBUTION THAT THESE
INDIVIDUALS MAKE. WE WANT TO
THINK ABOUT HOW WELL WE IDENTIFY
AND CONSIDER THE RISKS, REDUCE
THEM AS MUCH AS YOU CAN. AND SO
ONE THING THAT ALL OF YOU, MANY
OF YOU NOTED IS, LIKE, WHAT IS
REDUCE RISK MEAN IN THIS
CONTEXT? AND IT PICKS UP A
LITTLE ON WHAT JILL WAS
MENTIONING BEFORE. RIGHT?
INCLUDING SOMEONE WHO IS
UNINSURED MAY PUT THEM AT
FINANCIAL RISK. RIGHT? WHEN
SOMEONE IS UNINSURED THAT MEANS
THEY DON'T HAVE HEALTH
INSURANCE, RIGHT? AND FOR THOSE
OF YOU WHO HAVE EVER VISITED AN
EMERGENCY FOR EXAMPLE WHEN I
HAVE HEALTH INSURANCE, YOU'RE
GOING TO PAY SOME PORTION OF
THAT BILL. IF YOU DON'T HAVE
HEALTH INSURANCE AND AS A SIDE
NOTE, RIGHT, THOSE RATES ARE
NEGOTIATED WITH THE INSURANCE
COMPANIES, LARGE ACADEMIC
MEDICAL CENTERS OR HEALTH
SYSTEMS GET DISCOUNTS ON THAT
RATE. BUT IF YOU'RE A REGULAR
OLD PERSON WHO DOES NOT HAVE
HEALTH INSURANCE, YOU ARE PAYING
THE FULL AMOUNT OF THE CARE
PROVIDED. SO YOU MAY END UP
PAYING MUCH MORE WHEN YOU
COMPARE THE TWO COSTS FROM
SOMEONE WHO IS INSURED AND WHO
IS NOT. THAT'S A GENERAL
INJUSTICE IN OUR SYSTEM BUT
ENROLLING SOMEONE WHEN THERE'S
AN EXPECTATION THAT THERE IS
GOING TO BE STANDARD OF █CARE AD
SOMEONE'S GOING TO HAVE TO PAY
FOR IT. IT'S GOING TO BE
OUT-OF-POCKET FOR THAT
PARTICIPANT. SO ARE WE PUTTING
THEM AT FINANCIAL RISK OF
INCLUDING THEM? AND THEN WE
NEED TO BALANCE THAT ALONG WITH
THE PHYSICAL SOCIAL-EMOTIONAL
RISK OF BEING IN THE STUDY AND
THEN WHAT THE POTENTIAL BENEFITS
ARE. SO MAYBE WE WOULD WANT TO
ENGAGE WITH A PERSON IN AN
INFORMED CONSENT CONVERSATION
ABOUT WHAT MIGHT HAPPEN IF THEY
ENROLL? WHAT ADDITIONAL
FINANCIAL RESPONSIBILITIES THEY
MIGHT HAVE. WHAT IS REQUIRED BY
THE STUDY? IS THAT THIS
PARTICULAR BASELINE CARE BE
COVERED? AND SO MAYBE THEY WANT
TO MAKE A DECISION TO ENROLL
DESPITE THAT RISK. IS IT
APPROPRIATE FOR "US" OR IF THE
CLINICAL STUDY TEAM TO MAKE THAT
DECISION ON BEHALF OF THE
INDIVIDUAL? AND THEN LASTLY AS
IT RELATE TO FAIR PARTICIPANT
SELECTION, AGAIN, WE'VE TALKED
ABOUT THIS, THE IMPORTANCE OF
SORT OF SHARING THE RISKS,
BURDENS AND POTENTIAL BENEFITS
OF PARTICIPATION ACROSS ALL
INDIVIDUALS AND SOCIETAL GROUPS
AS POSSIBLE. SO WE WORRY ABOUT
BOTH EXCLUSION AND INCLUSION.
INCLUSION AS IT MEANS SOMEONE
HAS AN OPPORTUNITY OR A
POSSIBILITY OF BENEFITTING AN
EXCLUSION IN TERMS OF WHETHER OR
NOT THAT PARTICULAR GROUP IS
TAKING ON TOO MUCH OF THE BURDEN
OF THE TRIAL AND/OR WON'T
BENEFIT AS A RESULT OF THEIR
PARTICIPATION THE GROUP FROM
WHICH THEY COME IS LESS LIKELY
TO BENEFIT FROM THE FINDINGS OF
THE STUDY. WE'LL TALK MORE
ABOUT THAT NEXT WEEK WHEN WE
TRANSITION TO INTERNATIONAL
ISSUES. SO JILL MENTIONED UNDUE
INDUCEMENT AND SO ONE MIGHT BE
CONCERNED THAT IF THE PERSON WHO
ISN'T INSURED AND WANTS TO
ENROLL AT ONE OF THE SITES WHERE
THE STANDARD OF CARE WILL BE
COVERED, IF, LET ME SAY THAT A
DIFFERENT WAY, AT THE OTHER
THREE SITES, THEY PLAN TO ENROLL
UNINSURED PARTICIPANTS AND THE
SITES WILL COVER THE STANDARD OF
CARE COSTS FOR THOSE
PARTICIPANTS. SO THE
PARTICIPANTS IN THOSE OTHER
THREE SITES WILL NOT BE AT
FINANCIAL RISK. THIS OTHER SITE
WANTS TO EXCLUDE ANYONE WHO IS
INSURED BECAUSE THEY DON'T WANT
TO COVER THAT COST. AND SO THE
ONE WORRY MIGHT BE FOR THE FOLKS
WHO ARE IN THOSE OTHER SITES OF
IS PROVIDING COVERING STANDARD
OF CARE FOR THE PERIOD OF THIS
TRIAL WHICH IS UP TO A YEAR IS
THAT SOMEHOW AN UNDO INDUCEMENT
FOR THOSE INDIVIDUALS? AND I
WOULD ARGUE THAT IT'S LIKELY
NOT. THAT IT'S RELATIVELY
MODEST AND WHEN WE TALK ABOUT
UNDUE INDUCEMENT AND JILL
MENTIONED THIS BEFORE, SOMETIMES
WE THINK ABOUT IT IN THE SETTING
OF IF YOU'RE TALKING ABOUT
PEOPLE WHO HAVE A CONDITION,
THESE FOLKS ARE ALL AT HIGH RISK
OF HAVING LONG COVID. WHEN
THERE IS A POTENTIAL BENEFIT FOR
INDIVIDUAL WHO IS ENROLL MAYBE
THAT'S ENOUGH.
AND THEN WE DON'T TRADITIONALLY
PAY THEM FOR THEIR PARTICIPATION
BUT IN THIS CASE, THERE IS GOING
TO BE FOR THOSE WHO ARE IN THOSE
OTHER SITES, SOME FINANCIAL
BENEFIT IN COVERING THE STANDARD
OF CARE PROCEDURES THAT THEY
OTHERWISE WOULDN'T RECEIVE,
RIGHT? BECAUSE THEY ARE
UNINSURED, THEY ARE AT RISK OF
LONG COVID. THEY ARE GOING TO
BE OUT IN THE COMMUNITY. MAYBE
THERE'S SOMETHING THAT IS OF
UNDUE INDUCEMENT FOR THEM. BUT
IN THE CONTEXT OF A -- THAT FROM
MY PERSPECTIVE AND I TALKED A
LITTLE ABOUT THIS BEFORE, UNDUE
INDUCEMENT SORT OF KICKS IN WHEN
THE BENEFIT THAT THE PERSON MAY
RECEIVE AND IN THIS CASE IT'S A
POTENTIAL BENEFIT FROM THE
COMPOUND AND SOME FINANCIAL
BENEFIT OF HAVING STANDARD OF
CARE AVAILABLE. OVERWHELMS
THEIR CONCERNS ABOUT THE RISK OF
THE INTERVENTION THAT THEY MAY
BE RANDOMIZED TO. SO ANOTHER
WAY TO SAY THAT IS THAT BECAUSE
THE COVERAGE OF THE STANDARD OF
CARE IS SO VALUABLE TO THEM, IT
WOULD SWAMP THEIR ABILITY TO
MAKE AN INFORMED DECISION ABOUT
ENROLLMENT BECAUSE THEY'RE GOING
TO UNDERESTIMATE OR DIMINISH THE
POTENTIAL RISK FOR THEM. AND
FROM MY PERSPECTIVE, I DON'T
THINK THAT PROVIDING STANDARD OF
COVERAGE FOR STANDARD OF CARE
FOR THOSE OTHER FOLKS IS
PROBLEMATIC. AND YOU GUYS
BROUGHT THIS UP. CATEGORICALLY
EXCLUDING UNINSURED PATIENTS
WOULD BE A DISPROPORTIONATE
RESPONSE TO UNDUE INDUCEMENT
CONCERNS AND WOULD LIKELY
VIOLATE THAT PRINCIPLE ABOVE
THAT WE TALKED ABOUT IN TERMS OF
FAIR PARTICIPANT SELECTION. SO
I WANT TO END WITH SORT OF A
MORE META QUESTION IN THINKING
ABOUT CLINICAL TRIALS AND THE
ETHICS OF CLINICAL TRIALS.
SOMETHING THAT AGAIN, I'M GOING
TO SORRY, IT HAS TO RECONNECT
ITSELF. MY LAST QUESTION, IF
WE'RE THINKING ABOUT FAIRNESS,
RIGHT? IS IT, SO, AGAIN, JUST
TO REMIND YOU, WE HAVE THREE
SITES THAT ARE GOING TO ENROLL
PEOPLE WHO ARE UNINSURED OR
REGARDLESS OF THEIR INSURED
STATUS AND ANOTHER SITE THAT
WANTS TO EXCLUDE THEM. NOW IS
THAT FAIR TO OTHER PARTICIPANTS?
RIGHT? SO ANOTHER WAY TO THINK
ABOUT THAT IS, IF COVERING THAT
COST FOR THOSE WHO HAVE
INSURANCE OR CHARGING INSURANCE
IS COVERING THE COST FOR THOSE
WHO ARE UNINSURED UNFAIR TO THE
LARGER PARTICIPANT POPULATION?
AND/OR IS KOVRPING IT --
COVERING IT AT ONE SITE UNFAIR
TO THE OTHER SITES.
I MENTION THESE JUST IN THE
SENSE THAT YOU GENERALLY WHEN
WE'RE THINKING ABOUT THE CONDUCT
OF CLINICAL TRIALS, WE'RE
THINKING, YOU KNOW, ABOUT THE
CLINICAL TRIAL ITSELF. WE'RE
THINKING ABOUT THE RISK TO
PARTICIPANTS, THE BENEFITS TO
PARTICIPANTS, MAKING SURE THAT
IS BALANCED, CONSIDERING
INFORMED CONSENT. INCREASING
THE LIKELIHOOD THAT PEOPLE WILL
UNDERSTAND. SOMETIMES WE HAVE
TO LOOK AT HOW THE TRIAL ITSELF
IS BEING DESIGNED. AND HOW
DIFFERENT SITES OR DIFFERENT
POLICIES OR DIFFERENT CRITERIA
MIGHT INVITE DISPARITIES OR
INEQUALITIES AMONG SITES AND/OR
PARTICIPANTS AND SHOULD IT BE A
GOAL THAT EVERYONE IS DOING THE
SAME THING. IS THAT A
REASONABLE THING FOR US TO THINK
ABOUT WHEN WE'RE EVALUATING A
CLINICAL TRIAL. I DON'T THINK
WE -- SO I WILL SAY FROM MY
EXPERIENCE OF BEING ON MANY
IRBS, I AM NOT SURE THAT THE
AVERAGE INSTITUTIONAL REVIEW
BOARD THINKS AS MUCH AS THEY
SHOULD IN SOME CASES ABOUT
WHETHER OR NOT SOME OF THESE
ARRANGEMENTS ARE FAIR OR NOT.
AND SO I AM WONDERING, I WOULD
LIKE TO JUST OPEN THE DISCUSSION
AND FOLKS WHO ARE HERE ARE
WELCOME TO GO TO THE MICS AND/OR
FOLKS OUT IN VIDEO LAND CAN POST
RESPONSES TO MY QUESTION ON THE
DISCUSSION BOARD OF WHAT --
IS -- WHETHER OR NOT TO INCLUDE
PARTICIPANTS WHO ARE UNINSURED.
SHOULD WE SAY IN A SYSTEM THAT
IS ALREADY UNFAIR THAT IT WOULD
BE INAPPROPRIATE TO ALWAYS OR
CONSIDER TO EXCLUDE PEOPLE WHO
ARE UNINSURED? OR DO WE SAY,
WELL, GIVEN THAT OUR SYSTEM IS
BY DEFINITION UNFAIR WE
SHOULDN'T DOUBLY PLACE A BURDEN
ON THOSE WHO ARE UNINSURED AND
THEREFORE SHOULD MAKE SURE WE
INCLUDE THEM AS OFTEN AS WE CAN.
DOES ANYBODY HAVE ANY SORT OF
OPINION OR THOUGHTS ABOUT THAT?
AND I WILL MENTION THIS IS A
UNIQUE ISSUE FOR US IN THE
UNITED STATES IN THAT IT'S NOT
THE CASE THAT EVERYBODY HAS
ACCESS TO HEALTH CARE. ANOTHER
WAY OF SAYING THAT WOULD BE IS
WE DON'T HAVE TO HAVE THIS
CONVERSATION IN OTHER SETTINGS
WHERE BASIC HEALTH CARE IS
PROVIDED TO EVERYONE. RIGHT?
ONE WAY TO ADDRESS THE ETHICAL
CONCERN IS TO SAY, WELL, LET'S
MAKE SURE EVERYBODY'S INSURED.
WE CAN'T DO THAT. SO THEN WE
HAVE TO SORT OF MANAGE IS IT
OKAY, SHOULD WE ALWAYS INCLUDE
OR SHOULD WE EXCLUDE? AND WHAT
WOULD THE JUSTIFICATIONS FOR
THAT BE? ANY THOUGHTS? DANIEL,
YOU'RE KIND OF MOVING TOWARDS
THE MIC? WELL, IF YOU WOULDN'T
MIND GO TO THE MIC SO FOLKS OUT
IN THE WORLD CAN HEAR YOU A
LITTLE BETTER.
>> AUDIENCE: YEAH I CAN SEE
MAYBE AN ARGUMENT FOR EXCLUDING
THE UNINSURED IF THE COSTS OF
INCLUDING THEM WOULD
EXTERNALIZED SPECIFICALLY ONTO
THE INSURED PARTICIPANTS LIKE IF
THEY RECEIVED LESS COMPENSATION
BECAUSE MORE MONEY HAD TO BE
GIVEN TO, LIKE, PAY FOR THE CARE
OF THE INUNINSURED BUT EVEN THEN
IT SEEMS LIKE THE OTHER SIDE OF
JUST -- OF THAT IS YOU HAVE TO
EXCLUDE A BUNCH OF UNINSURED
POSSIBLE PARTICIPANTS AND THAT
JUST SEEMS TO BE AN OVERRIDING,
LIKE, ISSUE.
THAT JUST SEEMS PARTICULARLY
DISCRIMINATORY TO ME SO YEAH, I
THINK IT WOULD MAKE MORE SENSE
TO JUST HAVE, LIKE, IDEALLY SOME
SORT OF INSTITUTION, AT LEAST IN
THIS CASE, YOU KNOW, SUBSIDIZE
AND PAY FOR THE UNINSURED
MEDICAL CARE AND I GUESS EVEN
MORE IDEALLY WE WOULD HAVE A
DIFFERENT INSURANCE REGIME WHERE
IT'S NOT PRIVATE IN SUCH A WAY
IT WORKS WHERE PEOPLE ARE
UNINSURED.
>> HOLLY: FROM THE PERSPECTIVE
OF AN ACADEMIC MEDICAL CENTER,
WHEN I WAS AT HOPKINS THE SCHOOL
OF MEDICINE WOULD NOT ROUTINELY
EXCLUDE INDIVIDUALS BASED ON
THEIR INSURANCE STATUS. WHAT
THEY WOULD DO IS ONCE SOMEONE IS
IDENTIFIED AS ELIGIBLE AND
INSURANCE WAS HIGHLIGHTED AS A
CONCERN, THEY WOULD BASICALLY
SEND THE PERSON TO THE SOCIAL
WORKER WHO IS IN CHARGE OF
MAKING SURE THEY'RE RECEIVING
ALL THE BENEFITS THEY'RE
ELIGIBLE FOR. SOMEONE WHO IS
NEVER ENROLLED IN MEDICAID OR
LIVES IN A STATE LIKE MARYLAND
WHERE THERE ARE SAFETY NET
PROGRAMS THAT INDIVIDUALS CAN
APPLY FOR TO GET THEIR HEALTH
CARE INSURED. SO THAT'S, I
THINK THAT'S ONE WAY, IT DOESN'T
ELIMINATE THE PROBLEM, RIGHT?
THERE MAY BE SOME INDIVIDUALS,
RIGHT, WHO JUST ARE NOT ELIGIBLE
FOR -- THEY MAKE TOO MUCH OR
THEY'RE MALE OR THEY'RE NOT
PREGNANT OR ALL SORTS OF
REASONS. THEY MAY NOT QUALIFY
FOR ANY OF THE PUBLIC BENEFITS
AVAILABLE TO THEM THAT THEY WILL
BE UNINSURED. THEY DON'T HAVE A
JOB. MOST PEOPLE GET INSURANCE
THROUGH THEIR JOBS. SO THAT'S A
WAY OF AT LEAST TRYING TO CLOSE
THAT GAP. AND THE OTHER THING
TO THINK ABOUT, I MEAN, MY --
THE MOST EXPERIENCE I HAVE AS IT
RELATE TO INCLUSION OF
INDIVIDUALS WHO ARE UNINSURED IS
RAE
-- EARLIER IN THE HIV EPIDEMIC,
THERE WERE TRIALS THAT WERE OPEN
BACK THEN.
YOU WERE RANDOMIZED TO A PLACEBO
OR AN ACTIVE COMPOUND, YOU KNOW,
AS TIME WENT BY, THERE WOULD BE
MANY FEWER PLACEBO CONTROLLED
TRIALS BUT STILL TRIALS THAT
TESTED THE STANDARD TOWARDS THE
NEW NOVEL THING. THAT GROUP BY
DEMOGRAPHICALLY WERE MUCH LESS
LIKELY TO BE INSURED THAN THE
AVERAGE PERSON. IN TERMS OF
THEIR ABILITY TO QUALIFY FOR
DISABILITY ON MEDICARE, THEIR
ABILITY TO HOLD DOWN A JOB, ET
CETERA. SO, YOU KNOW, A BLANKET
EXCLUSION OF EXCLUDING THOSE
INDIVIDUALS MEANT THAT THEY
WOULD BASICALLY BE WITHOUT
HEALTH CARE FOR A PERIOD OF
TIME. OR THEY WOULD EXTEND THE
TIME WITH WHICH THEY DIDN'T HAVE
ACCESS TO HEALTH CARE. SO
REALLY SAW THIS AS AN
OPPORTUNITY TO HAVE STABLE
HEALTH CARE. WHICH, AGAIN,
ETHICALLY I FIND PROBLEMATIC BUT
I'M NOT GOING TO SAY AND
THEREFORE WE SHOULD EXCLUDE THEM
BECAUSE IF THAT'S THE ONLY WAY
TO HAVE ACCESS TO STABLE HEALTH
CARE THERE'S A TRADE-OFF THERE.
RIGHT? AND MY INCLINATION IS TO
SAY IF THAT'S THE ONLY WAY TO
HAVE ACCESS IT SEEMS, GIVEN THE
BACKGROUND CONDITIONS, GIVEN THE
CONTEXTUAL CONDITIONS IT SEEMS,
YOU KNOW, NOT ONLY REASONABLE
BUT IMPORTANT TO FACILITATE
ENROLLMENT IN CLINICAL TRIALS
AND ON THE FIRST DAY OF CLASS
CHRISTINE SHOWED, THERE WERE A
SERIES OF ERAS OF CLINICAL
RESEARCH AND ONE OF THOSE ERAS
WAS THE EARLY ADVOCACY FOR HIV
AND THE SIGNS THAT MANY WOULD
HOLD HERE AT THE NIH AND FDA IS
CLINICAL TRIALS IS HEALTH CARE.
RIGHT? AND SOME PEOPLE SAID,
WELL, YOU KNOW, THAT SHOULDN'T
BE THE CASE, RIGHT? LIKE
CLINICAL -- YOU SHOULDN'T THINK
OF CLINICAL TRIALS AS HEALTH
CARE, THAT'S A THERAPEUTIC
MISCONCEPTION BUT FOR THEM IT
WAS LITERAL IN THE SENSE OF
HAVING ACCESS TO A CLINICAL
TRIAL MEANS THAT I MIGHT HAVE
ACCESS TO SUSTAINABLE HEALTH
CARE. SO, THAT'S IT FOR ME
TODAY. WE ARE GOING TO RETURN
AT 10:10. I'M VERY EXCITED TO
HAVE A GROUP FROM THE PATIENT
ADVISORY GROUP FROM THE CLINICAL
CENTER WHO WILL THEIR EXPERIENCE
OF BEING PARTICIPANTS OF TRIALS
HERE AT THE CLINICAL CENTER. SO
WE WILL REGROUP AT 10:10. THANK YOU.
>> WE ARE GETTING GUESTS FROM A
DISTANCE ON THE SCREEN. SO
FIRST I WANTED TO WELCOME AND
THANK OUR MEMBERS FROM THE
PARTICIPANT ADVISORY GROUP FOR
JOINING US TODAY MY GOAL, OUR
GOAL IS TO GIVE YOU INSIGHT INTO
WHAT IT IS LIKE TO BE A
PARTICIPANT IN A RESEARCH
PROTOCOL HERE AT THE CLINICAL
CENTER AND I'M GOING TO BE A BIT
OF AN MC AND ASK FOLKS
QUESTIONS. AND THEN WE WILL
OPEN IT UP FOR QUESTIONS FROM
THOSE OF YOU WHO ARE HERE IN THE
AUDIENCE. AND THOSE OF YOU WHO
ARE JOINING US BY VIDEO SO FIRST
I WANT TO GO AROUND THE ROOM,
WE'LL CALL IT. AND HAVE YOU
EACH INTRODUCE YOURSELVES TO THE
GROUP AND THEN WE'LL GET INTO
QUESTIONS ABOUT YOUR EXPERIENCE.
>> HELLO, MY NAME IS ELLEN AND I
HAVE BEEN A PATIENT AT NIH SINCE
2001. SO A VERY LONG TIME. I
WAS ELIGIBLE FOR A PROTOCOL
BECAUSE I HAD SEVERE
HYPOGLYCEMIA UNAWARENESS WHERE I
WOULD PASS OUT WITH NO WARNING
FROM LOW BLOOD SUGAR. I'VE BEEN
A DIABETIC SINCE I WAS 13. A
MERE TEN YEARS AGO. AND SO I
KNEW WHAT A LOW BLOOD SUGAR WAS
LIKE. BUT I KIND OF LOST THE
ABILITY TO SENSE THAT AND THEN I
WOULD JUST CRASH AND SOMETIMES
IT WAS WHEN I WAS DRIVING THE
CAR AND I ALWAYS KNEW TO PULL
OVER AND STOP THE CAR AND THE
EMERGENCY PEOPLE WOULD BE THERE
TAKING ME OUT OF THE CAR, TAKING
ME TO THE HOSPITAL AND, YOU
KNOW, IT'S NOT A GOOD THING WHEN
THE EMERGENCY PEOPLE KNOW YOU ON
A FIRST NAME BASIS. THEY HAD
COME SO MANY TIMES TO MY HOME OR
TO MY OFFICE WHEN I WAS WORKING.
SO I STARTED TO LOOK INTO
RESEARCH TO FIND OUT WHAT WAS
GOING ON AND MY FATHER HEARD
ABOUT AN EYE LET CELL TRANSPLANT
ON THE NEWS AND SO HE CALLED ME
RIGHT UP AND SAID ELLEN YOU GOT
TO CHECK THIS OUT.
SO I STARTED TO CHECK IT OUT AND
I DID GET AN EYE LET CELL
TRANSPLANT IN 2001. DR. DAVID
HARLAN WAS MY PHYSICIAN. HE WAS
HERE AT NIH FOR MANY YEARS.
HE HAS SINCE MOVED ON. I HAVE
SINCE MOVED ON TOO. I HAD
FUNCTIONING EYE LETS THAT THEY
TOOK THE BETA CELLS WHICH ARE
THE LITTLE TINY CELLS THAT
PRODUCE INSULIN IN YOUR
PANCREAS, THEY TOOK THOSE FROM A
CADAVER AND PUT THEM INTO MY
LIVER. THEY'D LIVED HAPPILY IN
MY LIVER FOR ABOUT TEN YEARS SO
I NO LONGER HAD TO TAKE SHOTS
AND NO LONGER PASSING OUT
WITHOUT WARNING SO THAT WAS THE
CRITICAL PART AND FOR ME THAT
WAS THE SUCCESS OF THE CLINICAL
TRIAL. SO I THANK ALL OF YOU
WHO ARE INVOLVED IN RESEARCH
BECAUSE WITHOUT THAT ADVANCES
LIKE THIS COULD NOT HAPPEN. AND
THE OBJECT OF THE PROTOCOL WAS
NOT PROOF OF PURCHASE LIKE AT
THE STORE BUT A PROOF OF
CONCEPT. SEE MY CONCEPT WAS
GONE. PROOF OF CONCEPT AND THEY
DID PROVE THAT THAT CONCEPT DID
WORK. I WAS THE FIFTH ONE AT
NIH TO HAVE IT AND THE LONGEST
ONE TO HAVE FUNCTIONING EYE
LETS. I NO LONGER HAVE
FUNCTIONING EYE LETS BUT THAT'S
OKAY. I'M -- THE TECHNOLOGY HAS
IMPROVED SO MUCH SINCE I FIRST
STARTED COMING HERE. THAT I NOW
HAVE AN INSULIN PUMP AND A
CONTINUOUS GLUCOSE MONITOR SO MY
DIABETES IS VERY GOOD CONTROL.
MY A 1C IS ABOUT 6.7 NOW SO I AM
PLEASED WITH THAT.
AND MY ENDOCRINOLOGIST IS VERY
PLEASED WITH THAT. THAT'S MY
BRIEF HISTORY.
>> THANK YOU AND WE'LL COME BACK
WITH OTHER QUESTIONS.
>> GOOD MORNING I WANT TO START
AND SAY THANK YOU GUYS FOR BEING
DEDICATED TO RESEARCH BECAUSE
YOU GUYS ARE HELPING US OUT A
LOT I HAVE BEEN A PATIENT AT THE
NIH FOR TWELVE YEARS. IT'S HARD
FOR ME TO PROCESS THAT BUT IT
GOES BY REALLY FAST. OFF RARE
EYE DISEASE IT'S PROGRESSIVE AND
I'M SEEN AT THE NEI. I'M NOT
ENROLLED IN A CLINICAL TRIAL BUT
THEY LIKE TO COLLECT DATA ON ME
TO SEE HOW THE PROGRESSION OF MY
VISUAL IMPAIRMENT IS GOING IT'S
BEEN AN INTERESTING RIDE BUT I
CAN SAY IT'S NOT NECESSARILY
BEEN A ROLLER COASTER FOR ME IN
THIS EXPERIENCE IT'S BEEN A
STEADY TRAIN RIDE SO, YEAH.
STILL HERE, STILL TRYING TO
FIGURE OUT IF THEY CAN FIGURE
OUT WHAT MY EYE DISEASE IS.
SO....
>> THANK YOU. LIAM, WHY DON'T
WE GO TO YOU.
>> YEAH, SO, I'VE BEEN AN NIH
PATIENT FOR ABOUT FOUR YEARS NOW
OR -- NO, NO, TWO YEARS, I'VE
BEEN AN NIH PATIENT NOW FOR TWO
YEARS AND I CURRENTLY GO TO
COLLEGE AS A JUNIOR. BUT THE
RARE DISEASE THAT I HAVE IS
CALLED CLASSIC CONGENITAL
HYPERPLASIA WHICH BASICALLY
MEANS I DON'T PRODUCE CORTISOL
AND I JOINED A CLINICAL TRIAL AT
THE SAME TIME BACK IN 2022. YOU
KNOW, IN ORDER TO HOPE --
BASICALLY THE GOAL WAS THAT YOU
WANT TO REDUCE THE STEROID USAGE
THAT I CURRENTLY TAKE BECAUSE
LONG-TERM STEROIDS IS PRETTY BAD
SO THE GOAL WITH THE MEDICATION
IS TO REDUCE THAT STEROID USAGE
AND, YOU KNOW, OVERALL I WOULD
SAY THE TRIAL'S BEEN GOING
PRETTY WELL. I MEAN, THAT'S
PRETTY MUCH IT TO BE HONEST. I
DON'T HAVE MUCH ELSE TO SAY.
>> GREAT, WELL WE'LL GET TO SOME
DETAILS WHEN WE GO AROUND WITH
SOME OTHER QUESTIONS. NINA,
PLEASE GO AHEAD AND INTRODUCE
YOURSE
YOURSELF.
>> HI, EVERYONE, I'M NINA AND
I'M TALKING TO YOU FROM NEBRASKA
TODAY. SO I'M HAPPY TO BE HERE.
THIS IS REALLY EXCITING
DISCUSSION. AND THIS IS AN
IMPORTANT DISCUSSION AS WELL. I
HAVE A RARE DISEASE CALLED
GENERAL SIN'S CON DROE DYSPLASIA
AND I DIDN'T GET DIAGNOSED UNTIL
I -- HAD PASSED IT DOWN TO MY
TWO CHILDREN AND I WAS 32 YEARS
OLD WHICH AFFECTS LESS THAN 30
PEOPLE WORLDWIDE.
SO I AM ONE OF THE OLDEST WITH
THIS CONDITION STILL LIVING.
AND I MOVED TO AMERICA FROM THE
MIDDLE EAST. WHEN WE FOUND OUT
THAT THERE WAS PROBABLY SOME
GLIMMER OF HOPE THAT WE COULD
TREAT THIS CONDITION I'VE BEEN
AT THE NIH, FAIRLY NEW, IN
COMPARISON TO SOME OF MY OTHER
COLLEAGUES HERE ON THE PANEL.
I'VE BEEN HERE SINCE 2022. AND
WE ARE UNDER THE CARE OF A
DOCTOR AND WILL BE PART OF A
CLINICAL TRIAL AND I WILL BE THE
FIRST PATIENT TREATED WITH AN
INVERSE AGONIST FOR MY
CONDITION. PRETTY SOON HERE.
SO WHAT WE ARE DOING IS WORKING
ON THE NATURAL HISTORY STUDY OF
THIS VERY ULTRA RARE DISEASE
THAT HAS HARDLY ANY PATIENTS.
SO IT'S A VERY DIFFERENT
APPROACH BECAUSE THERE'S SO MANY
UNKNOWNS. AND SO WE DON'T
REALLY HAVE A CLEAR PATH AS, YOU
KNOW, WHAT RESEARCH SHOULD LOOK
LIKE IN AN IN A ONE OR A FEW
WHEN THERE'S NO HISTORY BUT
GIVEN THE DISEASE IS PROGRESSIVE
AND DEBILITATING IT'S URGENT.
SO THE FACT THAT WE GOT TO MOVE
QUICKLY AND AT THE SAME TIME
UNDERSTAND THIS DISEASE WHILE
ALSO GETTING PATIENTS INTO THE
CLINIC AND MANY OF THEM ARE
CHILDREN. I THINK IT REALLY
SETS UP FOR NOT JUST A
CHALLENGING RESEARCH STUDY BUT
ALSO ONE THAT REQUIRES A LOT OF
COLLABORATION BETWEEN PATIENT
AND RESEARCHER ON A JOURNEY OF
DISCOVERY AS WELL AS FINDING
TREATMENTS FOR A POPULATION THAT
IS SO SMALL THAT COULD BE
CONSIDERED TOTALLY
INSIGNIFICANT. YET, SO
IMPORTANT, BECAUSE IT WILL LEAD
US TO IMPORTANT DISCOVERIES THAT
WILL HELP GENERAL DISEASES. SO
I AM HAPPY TO BE PARTICIPATING
IN THIS DISCUSSION TODAY BECAUSE
EVEN IN THIS TWO YEARS, I'VE
LEARNED SO MUCH AND IN THIS
PROCESS AND I'M REALLY EXCITED
FOR THE WORK THAT IS GOING TO
COME, NOT JUST FROM THIS JOURNEY
BUT ALSO FROM THE DISCUSSION
HERE TODAY.
>> THANK YOU, ALL, SO MUCH AND
MAYBE I WILL COME BACK TO YOU,
NINA TO ASK, SOUND LIKE YOU'RE
ON THE EDGE OF ENROLLING IN AN
ALREADY UNIQUE TRIAL AND I'M
WONDERING IF YOU CAN SHARE SOME
OF THE THINGS THAT YOU'VE BEEN
THINKING ABOUT OR HAVE THOUGHT
ABOUT IN ANTICIPATION OF
ENROLLING IN A TRIAL THAT IS
BASICALLY THE FIRST, SOUNDS LIKE
IT'S GOING TO BE THE FIRST IN
HUMAN TRIAL OF THIS
INTERVENTION.
>> YES, SO THERE ARE A COUPLE OF
THINGS. ONE IS THAT, YOU KNOW,
WHEN YOU ARE SO RARE, AND WHEN
YOU LIVED WITH A DISEASE FOR SO
LONG AND HAD NO IDEA WHAT IT WAS
AND NOW SUDDENLY YOU KNOW, FAST
FORWARD AND YOU ARE ON THE VERGE
OF A TREATMENT. I MEAN, THE
SIGNIFICANCE OF THAT IS
SOMETIMES SO OVERWHELMING THAT
YOU DON'T REALLY HAVE A MINUTE
TO REALLY WORNED WHAT YOU'VE
DONE, RIGHT? SO WHEN WE GOT A
DIAGNOSIS, THE FIRST IDEA WAS
LIKE, HOW DO WE GET A TREATMENT?
AND WHEN I READ UP ABOUT THE
CONDITION, IN FACT THERE WAS
NOTHING WRITTEN, IT WAS JUST ONE
LINE IN ONE RESEARCH PAPER THAT
SAID WE COULD POSSIBLY TURN OFF
THE, YOU KNOW, I HAVE AN
OVERACTIVE PTH RECEPTOR SO WE
COULD POSSIBLY TURN THAT OFF BY
CREATING AN INVERSE AGONIST AND
IT WAS ONE LINE IN ONE RESEARCH
PAPER AND WE PACKED OUR BAG TO
FIND THE RESEARCHER THAT HAD
WRITTEN THAT PAPER AND THEN
TOGETHER WITH HIM AND WE STARTED
THE FOUNDATION FOR THIS DISEASE
CALLED THE GENERAL SIN'S
FOUNDATION AND THEN IT WAS A LOT
TO FIND OUT IF WE COULD GET
FIRST THE FUNDING TO BE ABLE TO
MOVE THIS INTO TREATMENT BECAUSE
IN RARE DISEASES, YOU KNOW SH,
THERE'S JUST NOT ENOUGH
RESOURCES, NOT ENOUGH FUNDING.
AT FIRST THERE WERE ONLY THREE
PATIENTS ME AND MY TWO BOYS.
AND IT'S OUR LIVES DON'T MATTER.
THAT'S BASICALLY WHAT THEY'RE
SAYING. YOU HAVE ALL THIS
SCIENCE AND WE DON'T MATTER. IT
WAS A JOURNEY TO CHANGE THAT
NARRATIVE. AND ONCE WE GOT A
RESEARCHER ON BOARD AND
DR. SMITH FROM THE NIH I THINK
THAT WE DIDN'T, IT WAS VERY
UNIQUE BECAUSE I DIDN'T HAVE
TIME TO THINK ABOUT, OKAY, AM I
GOING TO GET INTO THIS RESEARCH
INTO AND IS SOMETHING NEGATIVE
GOING TO HAPPEN? I HAD TO JUMP
IN WITH HOPE. HOPE AND TRUST
AND THESE ARE IMPORTANT FOR ANY
RESEARCH. I CAN'T SEE THE ROOM
BUT TO THOSE IN THE ROOM, TRUST
IS INCREDIBLY IMPORTANT. WE
DON'T GET INTO RESEARCH BECAUSE,
YOU KNOW, WE THINK THAT, YOU
KNOW, THERE IS SOME, YOU KNOW,
YES, IN MANY WAYS WE ARE
THINKING, WOW, WE'RE GOING TO
CHANGE SCIENCE AND WE'RE HERE
GIVING OURSELVES FOR RESEARCH
BUT YOU'VE GOT TO ALSO
UNDERSTAND THAT WE'RE GIVING
OURSELF TO THE TRUST THAT WE
HAVE IN YOU. AS A PARTNER. AS
AN EQUAL IN THIS PROCESS,
ESPECIALLY IN ULTRA RARE
DISEASES. OR IN RARE DISEASES.
THAT IS WHAT PROPELS US SO THE
WHOLE PROCESS HAS BEEN I HAVE TO
SAY PRETTY INCREDIBLY POSITIVE.
YOU KNOW, WE'VE GONE THROUGH THE
INFORMED CONSENT PROCESS. AYE
HAD MY CHILDREN HAVE TO CONSENT
AS WELL THAT PROCESS HAS BEEN
PRETTY SEAMLESS WE HAVE
UNDERSTOOD RIGHT FROM THE
BEGINNING WHAT THAT PROCESS HAS
BEEN LIKE. BUT, YOU KNOW, IT'S
ALSO UNDERSTANDING THAT RISK
BENEFIT RATIO. RIGHT? WHAT AM
I GOING TO DO? WHAT AM I GOING
TO COMMIT TO IN ORDER TO MAKE
SURE THAT MY CHILDREN AND OTHERS
WITHIN MY COMMUNITY WILL HAVE A
TREATMENT? WHAT SAFEGUARDS ARE
IN PLACE TO PROTECT MY CHILDREN
FROM ANY PHYSICAL EMOTIONAL OR
PSYCHOLOGICAL HARM? THAT IS
GOING TO HAPPEN DURING THE
STUDY? SO I THINK THAT'S
DEFINITELY GOING THROUGH MY MIND
BUT PREDOMINANTLY IT WAS MY
TRUST IN DR. SMITH, IN THE
CLINICAL CENTER, EVERY PERSON I
CAME ACROSS AT THE CENTER WAS
POSITIVE, WAS ENCOURAGING, WAS
HELPFUL. YOU KNOW, THEY WERE
ALL FASCINATED BY OUR STORY.
WHICH THAT'S ANOTHER THING.
IT'S IMPORTANT FOR US TO
REMEMBER THAT THESE ARE HUMAN
BEINGS AND THEY COME WITH
STORIES AND BACKGROUNDS AND ALL
THESE HOPES AND, YOU KNOW, AND
FEARS AND SO REALLY BEGIN TO
DISMANTLE THAT THROUGH THE WHOLE
RESEARCH PROCESS AND BE THERE IN
A SUPPORTIVE MANNER, I THINK IS
REALLY PARAMOUNT.
>> GREAT, THANK YOU VERY MUCH.
SO I'D LIKE TO GO TO YOU NEXT,
LIAM, IF YOU CAN TELL US A
LITTLE BIT ABOUT, IT SOUND LIKE
A COUPLE OF YEARS AGO YOU HAD
THE OPPORTUNITY TO ENROLL IN THE
STUDY. IF YOU CAN TELL US A BIT
ABOUT WHAT WAS THAT LIKE, WHAT
WAS THINKING ABOUT THE
OPPORTUNITY TO ENROLL IN A
CLINICAL STUDY LIKE? WHAT WERE
YOU THINKING ABOUT?
>> YEAH SO THE STUDY ACTUALLY
WAS RIGHT BEFORE, I ACTUALLY
WENT TO NIH IN 2022 AND I WAS
GOING -- THIS WAS RIGHT BEFORE
COLLEGE LIKE ENTERING COLLEGE
AND BASICALLY THE GOAL OF THE
GOING TO THE NIH WAS FOR THEIR
LIFELONG STUDY OF CAH WHICH IS
JUST TO MONITOR PATIENTS, MALE
PATIENTS THROUGHOUT THEIR WHOLE
LIFE AND THAT WAS THE FIRST TIME
I STARTED LOOKING AT THE
CLINICAL TRIAL.
SEEING IF I WANTED TO JOIN, ET
CETERA. AND WE WERE GOING TO GO
THROUGH NIH WITH THE CLINICAL
TRIAL AND AL THAT POINT I
COULDN'T GET INTO NIH BECAUSE
ALL THE SPOTS FILLED UP.
FORTUNATELY MY SCHOOL HAD THE
CLINICAL TRIAL AND I COULD JOIN.
AND I LOOKED AT THE BIG
QUESTION, IS IT SAFE AND, YOU
KNOW, WHAT ARE THE RISKS OF THE
CLINICAL TRIAL? RIGHT? THAT
WAS PROBABLY BEFORE I WAS
CONSIDERING ENROLLING WHAT WAS
MY BIGGEST TWO CONCERNS BECAUSE
AT THAT TIME I WAS 18. AND I
WOULD STILL CONSIDER MYSELF A
YOUNG ADULT KID BUT, AND THEN
WHAT KIND OF MADE ME I GUESS
PUSH MYSELF OVER THE EDGE TO
JOIN WAS BASICALLY I KNOW THIS
SOUNDS KIND OF MESSED UP THE
FACT THAT THERE WERE OTHER KIDS
IN THE TRIAL MADE ME REALIZE OR
AT LEAST FEEL SAFE THAT, YOU
KNOW, IF THEY'RE TESTING ON KIDS
AT THIS POINT THEN THE DRUG IS
PROBABLY PRETTY SAFE OR I HOPE
IT IS AND IT WAS ALSO A PHASE
THREE TRIAL AT THAT POINT SO IT
HAD BEEN GOING ON FOR TWO TO
THREE YEARS NOW AND I WAS
ACTUALLY I THINK THE LAST PERSON
TO JOIN OF THE TRIAL LIKE I
JOINED NOVEMBER 2022 AND THEN IT
SHUT DOWN DECEMBER 1ST SO I WAS
ONE OF THE LAST PEOPLE. AND,
YOU KNOW, BASICALLY I STILL HAD
TIME TO BACK OUT, LIKE, UNTIL
JANUARY SO WHEN WE WERE JUST
KIND OF GETTING INTO THE MAKING
SURE MY LEVELS WERE, YOU KNOW,
QUALIFIED, ET CETERA I WAS STILL
THINKING ABOUT HOW SAFE IS IT?
I GUESS THAT'S ALWAYS A BIG
CONCERN OF MINE. IF I TAKE THIS
DRUG I WOULD BE ONE OF THE FIRST
PEOPLE TO TAKE THIS DRUG
THROUGHOUT MY LIFE SO WE DON'T
KNOW WHAT THE LONG-TERM
SITUATIONS ARE. SO I HAD
ANXIETY ABOUT THAT. BUT, YOU
KNOW, ONCE I ACTUALLY GOT ON THE
DRUG, I SAW THE BENEFITS OF IT,
LIKE, MY STEROID USAGE WENT DOWN
STRAIGHTAWAY. NOT STRAIGHTAWAY
BUT PRETTY SOON AND, YOU KNOW,
LIKE I JUST SAW A LOT OF THE
SIDE EFFECTS OF CAH ALSO GET
MINIMIZED BECAUSE I WASN'T ON AS
MUCH STEROIDS. SO THE ANXIETY
OF THE LONG-TERM IS NOT AS BIG
OF A DEAL TO ME BECAUSE, WELL, I
GUESS THE SHORT-TERM I'M SEEING
BENEFITS. AND I GUESS FOR ME
THAT WEIGHS, I GUESS THAT
OUTWEIGHS THE LONG-TERM STEROID
USAGE BASICALLY, I'M TRYING TO
THINK OF THE WAY TO SAY IT. THE
LONG-TERM STEROID USAGE
BASICALLY GOT, HOW CAN I SAY IT?
>> IT'S A TRADE-OFF, RIGHT?
BETWEEN IMAGINING BEING ON THOSE
STEROIDS FOR A LIFETIME VERSUS
GIVING THIS DRUG A TRY.
>> YES, YES, EXACTLY. THANK
YOU.
>> YEAH, SURE. SO WHY DON'T YOU
TELL US A LITTLE BIT ABOUT HOW
YOU FIRST ENROLLED IN THE TRIAL?
>> SURE. FOR ME, I WAS PROBABLY
AN UNDERGRAD AND I HAD A LOW
VISION SPECIALIST OUTSIDE WHO
DID -- HE WOULD COME TO THE NIH
AT THE NEI, THE NATIONAL EYE
INSTITUTE, ONCE A MONTH AND HE
THOUGHT I WAS REALLY STRUGGLING
GOING TO SKOCHOOL, READING BOOK
AND STUFF AND HE SAID MY HANDS
ARE TIED WHY DON'T WE TRY TAKING
YOU TO THE NIH AND FOR ME IT WAS
LIKE, YEAH, LET'S GO.
I WAS WILL BE AND EXCITED TO
COME, DEFINITELY. THAT FIRST
INITIAL DAY WHEN I CAME AND I
GOT TO MEET DR. Z, GOSH IT FEELS
LIKE YESTERDAY BUT IT WAS JUST
MY HOPE LIGHT JUST WAS GETTING
BRIGHTER AND BRIGHTER AND
BRIGHTER. AGAIN, KIND OF
HEARING WHAT NINA WAS SAYING IT
IS A RARE DISEASE THAT I HAVE.
IT WASN'T A RARE DISEASE AND
THERE'S A CLINICAL TRIAL I HOPE
ONE DAY I CAN BE THAT FIRST IN
HUMAN PROCESS. I AM IN THE
BACKS. I WANT PEOPLE TO BE
AWARE OF THIS BECAUSE THERE IS
SOMEWHERE OUT THERE THAT
PROBABLY HAS THE SAME DISEASE AS
ME. SO THAT IS WHAT MOTIVATED
ME TO GO SAYING HEY, THERE IS
NOT A LOT OF PEOPLE ON THE BOOKS
HAVING THE ISSUES I AM HAVING.
MAYBE I CAN BE THE FIRST ONE AND
HELP PEOPLE MOVE THIS DISEASE
FORWARD IN HELPING PEOPLE DOWN
THE ROAD.
>> ELLEN IT SOUNDS LIKE YOUR DAD
CALLED AND SAID, OH MY GOSH YOU
SHOULD GET IN THIS TRIAL, TELL
US A LITTLE BIT MORE ABOUT THAT
STORY.
>> YEAH, HE FIRST KNEW OF THIS.
AND SO THEN I STARTED TO DO
RESEARCH. YOU KNOW, AND I WENT
TO CLINICAL TRIALS AND FOUND
CLINICALTRIALS.GOV AND FOUND THE
ACTUAL CLINICAL TRIAL AND READ
ALL THE DETAILS AND MY DISEASE
IS NOT RARE, UNFORTUNATELY. BUT
MY PARTICULAR DIFFICULTY WITH IT
WAS RARE AND IT WAS AFFECTING ME
SO I WAS VERY CONCERNED ABOUT
THAT. BECAUSE I NEVER KNEW IF I
WAS GOING TO KILL SOMEONE ELSE
OR MYSELF. WHEN I WOULD PASS
OUT. AND I WOULD HAVE TO CHANGE
MY WHOLE LIFE AND NOT WORK
ANYMORE AND, YOU KNOW, SO I WAS
VERY CONCERNED ABOUT THAT. AND
THEN I FIGURED OUT ALL, READ
EVERYTHING ABOUT IT. AND YOU
KNOW IT WAS LIKE, WELL WHY NOT?
NOTHING ELSE HAS WORKED. I'VE
TRIED MANY THINGS. GONE TO MANY
DOCTORS. AND THEY JUST DIDN'T
KNOW. THEY DIDN'T KNOW HOW TO
WAKE MY BODY UP SO I KNEW WHEN I
WAS GOING TO GO LOW. AND THEY
THOUGHT THAT, YOU KNOW, SO I
TALK IT OVER WITH MY REGULAR
PRACTITIONER AND SHE SAID, WELL,
I THINK YOU SHOULD GIVE IT A
TRY. WHY NOT? AND IT WAS A
QUESTION OF WHY NOT. BUT THEN
IT WAS, LIKE, WELL, OF COURSE.
YOU KNOW, LIKE HERE I AM. LIKE
YOU SAID TOO, HERE I AM, LET'S
GO. AND I CAME FOR ALL THE
INTERVIEWS. AND ONE OF THEM,
YOU KNOW, THEY CONTINUALLY SAID
THROUGHOUT, YOU KNOW, AND YOU
CAN DROP OUT AT ANY POINT, YOU
KNOW. BUT I REALLY FELT I WAS A
MEMBER OF THE TEAM THAT IT WAS A
GROUP DECISION AND ANYTHING THAT
WENT ON WE ALL GOT TO TALK ABOUT
IT FIRST, YOU KNOW, AND I REALLY
FELT THAT IT WAS, I WAS THE
VALUABLE MEMBER OF THAT TEAM
BECAUSE IT WAS ME THEY WERE
GOING TO DO IT ON BUT THEY
REALLY RESPECTED WHO I WANTED TO
DO AND WHAT I THOUGHT WAS GOING
TO HAPPEN SO REALLY WAS EXCITED
ABOUT THAT. SO THAT'S WHEN I
SAID, YEAH, OKAY, LET'S GO
AHEAD. ONCE I APPROVED. ONCE I
WAS APPROVED THERE WAS NO
QUESTION, I HAD GOTTEN THAT FAR
AND THOUGHT NO, THERE'S NO
REASON FOR ME NOT TO CONTINUE
WITH THIS, YES, I AM GOING TO
CONTINUE. I'M NOT GOING TO DROP
OUT AT ANY PARTICULAR TIME. BUT
THEY REALLY EM NO SIZED THAT
THAT WAS AN OPTION. YOU KNOW.
SO I WAS VERY GRATEFUL THAT I
WAS ABLE TO PARTICIPATE IN THE
TRIAL.
>> GREAT, SO MAYBE I WILL STAY
WITH YOU. IF YOU CAN TELL US,
LIKE, IMAGINE, YOU KNOW, I OR
ANYBODY IN THE AUDIENCE HAS
NEVER PARTICIPATED IN A CLINICAL
TRIAL WHAT YOU SAY WHAT WAS THE
PROCESS LIKE? HOW OFTEN DID YOU
HAVE TO COME? WAS THERE
ANYTHING THAT HAPPENED THAT YOU
WAS UNEXPECTED?
>> I THINK THAT WAS UNEXPECTED
WAS HOW MANY TIMES I HAD TO
COME. I CAME FREQUENTLY. AND I
CAME TO NIH BEFORE I WAS EVEN
ACCEPT BLOOD THE TRIAL THERE
WERE MANY INTERVIEWS. YOU KNOW,
THERE WAS A SOCIAL WORKER
INTERVIEW TO FIND OUT IF I HAD A
SUPPORT NETWORK. THERE WAS ALL
THE CLINICIANS. YOU KNOW, AND
PEOPLE WOULD CHANGE THROUGHOUT
THE PROCESS TOO. SOME OF THE
FELLOWS WOULD COME AND GO AND I
WOULD GET TO KNOW THEM AND THEY
WOULD FOLLOW ME THROUGH AND
INITIALLY AS PART OF THE TRIAL I
HAD TO COME VERY FREQUENTLY TO
GET BLOOD WORK DRAWN. VERY
FREQUENTLY. AND I AM A VERY
HARD STICK. SO, YOU KNOW,
THEY'D SEE ME COMING IN
PHLEBOTOMY AND THEY WOULD GO
(GROANS) OH, HERE SHE COMES
AGAIN. I SAVED ALL THE WRAPS
AFTER YOU HAVE THE BLOOD DRAWN
AND WRAPPED THEM AROUND MYSELF
AND TOOK A PICTURE OF THAT AND
SEND IT TO THE WRAP PEOPLE AND
SAID THIS IS WHAT YOU CAN DO
WITH YOUR WRAPS. THAT'S HOW
MANY TIMES I WAS COMING TO THE
NIH TO GET MY BLOOD DRAWN SO I
THINK THAT WAS UNEXPECTED THAT I
DIDN'T REALIZE HOW MANY TIMES
AFTER THE ACTUAL TRANSPLANT THAT
I WOULD HAVE TO COME.
>> YEAH. YEAH. AND IT SOUNDS
LIKE YOU'RE IN A NATURAL HISTORY
STUDY. HOW OFTEN DO YOU COME?
>> I COME IN ONCE A YEAR AND
IT'S A FULL DAY FROM 8 A.M. TO 5
P.M. IT'S PRETTY LONG. I'M
CONSTANTLY DOING DIFFERENT TYPES
OF MACHINE EXAMS, TAKING
PICTURES OF MY EYES AND THE
FIRST DAY WHEN I CAME I JUST WAS
LIKE THIS ISN'T JUST A REGULAR
EYE DOCTOR VISIT IT WAS
BACK-TO-BACK TO BACK BUT
EMPHASIZING WHAT ELLEN WAS
SAYING THAT WHOLE TEAM, THEY
WERE WITH YOU IN THE MORNING ALL
THE WAY UNTIL THE END. IT'S
REALLY, THEY DON'T LEAVE YOU
ALONE, THEY'RE CONSTANTLY
CHECKING IN WITH YOU, MY DAD
ALWAYS COMES WITH ME. SOME
SMALL LITTLE THING OF GETTING A
LITTLE BROWN BAG THAT HAD A
BANANA, APPLE, CHEESE, JUST TO
GET US THROUGH THE DAY THAT MADE
THE EXPERIENCE SO MUCH BETTER
FOR US. AGAIN, IT'S LONG DAYS.
YOU'RE NOT NECESSARILY, LIKE,
YEAH, LET'S, YOU GET TIRED. BUT
THE TECHNICIAN, YOU BECOME -- I
BECAME SO CLOSE TO MY TECHNICIAN
I WOULD SEE HIM EVERY YEAR, IT
WAS UPDATES, WHAT'S GOING ON
WITH YOUR LIFE, HERE'S THIS NEW
MACHINE, DO YOU WANT TO TRY IT?
>> SURE. YEAH.
>> SO THOSE LITTLE THINGS MADE
THAT TEAM MORE PERSONABLE AND
THIS IS WHAT SHE LIKES TO DO
FIRST VERSUS WE'LL DO THIS STUFF
LATER. THAT MADE IT MUCH MORE
EASIER TO COME AND TO HAVE THOSE
LONG DAYS.
>> I THINK THAT IS THE GOOD
POINT IS THEY KNOW YOU AS A
PERSON AND RESPECT YOU AS A
PERSON AND YOU'RE A PART OF THE
TEA
TEAM.
>> LIAM, TELL US WHAT AN AVERAGE
STUDY VISIT IS LIKE FOR YOU.
>> FORTUNATELY I HAVE MY
CLINICAL TRIAL AT MY UNIVERSITY.
JUST LIKE RIGHT ACROSS THE
STREET. AND I MEAN, OVERALL, I
WOULD SAY THE EXPERIENCE WAS
GOOD. THE PROBLEM ALSO FOR ME
WAS THE BEGINNING WHERE THEY HAD
TO DO A BUNCH OF BLOOD DRAWS.
BECAUSE I AM A COLLEGE STUDENT I
AM MISSING CLASS BUT I AM GOING
IN FOR A BLOOD DRAW EVERY OTHER
WEEK AND THE ISSUE WOULD
BECOME -- WE WOULD GET THE BLOOD
DRAWN AND I WOULD SIT THERE FOR
TWO TO THREE HOURS AND I HAD TO
WAIT AND DO MY BLOOD AGAIN SO
THERE WAS A BUNCH OF DEAD SPACE
IN BETWEEN WHERE I WOULD JUST BE
SITTING THERE DOING NOTHING
EXACTLY, BUT I HAD TO DO THAT
BECAUSE OBVIOUSLY THE CLINICAL
TRIAL AND IT WAS JUST VERY TOUGH
BECAUSE, YOU KNOW, I HAD A BUNCH
OF CLASSES AND IT WAS FRESHMAN
YEAR SO I WAS STILL ADJUSTING TO
COLLEGE. KNEW UNFORTUNATELY
LIKE WHAT THE OTHERS SAID.
THEY TREATED YOU AS A TEAM
MEMBER.
I FELT THE SAME WAY. I ACTUALLY
WAS WORKING WITH THE DOCTOR THAT
I MET WHEN I WAS EIGHT YEARS OLD
AND THEN LEFT FOR LIKE TEN YEARS
AND I NEVER SAW HIM BUT I CAME
BACK AND HE WAS STILL THERE AND
NOW HE WAS DOING THE CLINICAL
TRIAL SO, YOU KNOW, IN THAT
SENSE AT LEAST I WAS ABLE TO,
YOU KNOW, RECONNECT WITH HIM AND
KIND OF JUST TALK ABOUT, LIKE,
WHAT HAS BEEN GOING ON THESE
PAST TEN YEARS WHICH WAS NICE.
YOU KNOW, WHEN YOU HAVE TO SIT
THERE FOR ABOUT TWO HOURS EVERY
OTHER WEEK. OVERALL THE
EXPERIENCE, I WOULD STILL DO IT
AGAIN. NOW THAT I'M LONGER IN
THE TRIAL, IT'S BEEN ABOUT A
YEAR AND A HALF, TWO YEARS. I
MEAN, I AM THERE FOR PROBABLY
LIKE 40 MINUTES AND THEN WE'RE
DONE. ALL WE HAVE TO DO IS DRAW
THE BLOOD AND, YOU KNOW, AGAIN,
AS WAS MENTIONED BEFORE THE
CLINICAL TRIAL DOES WORK, LIKE
MY STEROID USAGE WENT FROM 25
MILLIGRAMS TO 17 AND A HALF AND,
YOU KNOW, I FEEL GREAT. I DON'T
FEEL ANY SIDE EFFECTS OR ANY
RISK AT ALL AND I MEAN,
UNFORTUNATELY THE CLINICAL
TRIAL, THAT UNFORTUNATELY FOR ME
BECAUSE I WILL HAVE TO DO THIS
IN DECEMBER WHEN THE CLINICAL
TRIAL ENDS. WE'LL SEE.
>> NINA, IT SOUNDS LIKE SO FAR
YOU'VE BEEN IN A NATURAL HISTORY
STUDY AND SOUNDS LIKE YOUR SONS
AS WELL. TELL US A BIT ABOUT
WHAT THOSE VISITS ARE LIKE.
>> YEAH. ABSOLUTELY. SO I
THINK THAT THEY'RE UNIQUE
CHALLENGES FOR ADULTS AND FOR
CHILDREN. WE DEFINITELY, ALL OF
US TO BE ABLE TO DO THIS IS,
EXTREMELY A BLESSING. IT'S A
PRIVILEGE TO BE A PART OF THIS
BUT SOMETIMES WHEN YOU REFLECT
ON WHAT THE EXPERIENCE IS LIKE,
YOU KNOW, SOMETIMES WE, IT'S
OVERSHADOWED REALLY THAT
ANTICIPATION OF BEING PART OF
SOMETHING REALLY SPECIAL AND
GREAT THAT CAN HELP OTHERS.
SOMETIMES CAN BE OVERSHADOWED BY
THE REALITIES OF BEING IN A
STUDY AND THE STUDY ENVIRONMENT
PER SE. SO FOR US WE HAD TO BE
THERE A WEEK AND IT WAS PRETTY
GRUELLING IN THAT, YOU KNOW,
BECAUSE WE'RE -- AND I HAVE TO
PREFACE THIS BY THE FACT THAT
NOBODY HAS EVER STUDIED THIS
BEFORE. SO IT HAS BEEN A LOT OF
LET'S DO EVERY SINGLE TEST
POSSIBLE AND THEN BEGIN TO SORT
OF REFINE THOSE, RIGHT? AS WE
UNDERSTOOD YES IT DOESN'T IMPACT
THIS AREA OR YES, IT DOESN'T
REALLY -- OR YES IT DOES IMPACT
THIS AREA SO NOW WE NEED MORE
TESTS FOR THIS. IN A SENSE WE
REALLY ARE CHARTING A NEW PATH,
RIGHT? SO THAT CAN ALWAYS BE
DIFFICULT BECAUSE YOU HAVE A
SCHEDULE OF APPOINTMENTS AND
TESTS AND PROCEDURES THAT NOT
NECESSARILY ARE THE SAME. RIGHT
RIGHT? BECAUSE THE FIRST TIME
WE WENT THERE WE HAD A WHOLE
BATTERY OF TESTS. AND SOME WERE
A LITTLE BIT OF TESTS THAT WERE
MORE INTENSE THAN OTHERS AND I
THINK IT'S IMPORTANT FOR US AS
PATIENTS AND WHO KNOW OR
CONDITION THE BEST TO BE ABLE TO
SAY NO, I DON'T WANT THIS TEST.
THIS TEST DOESN'T MAKE SENSE.
BECAUSE WE ARE THE BEST PEOPLE
TO PROVIDE THAT INPUT. AND I
THINK IT'S IMPORTANT FOR THE
RESEARCH FINGER TO LISTEN AND
UNDERSTAND THAT AND TO
CONFERENCE WITH US. TO SIT
THERE AT THE TABLE AND PLAN IT
OUT SO LIKE I MENTIONED OURS IS
A UNIQUE RESEARCH PATH IT'S NOT
YOUR STANDARD SET OF TEST. IT'S
SO IMPORTANT FOR US TO
COMMUNICATE AND TO HAVE
CONVERSATION AROUND WHAT THIS
TEST DAY IS GOING TO LOOK LIKE,
WHAT MY SCHEDULE IS GOING TO BE
AND WHETHER THIS IS IMPORTANT OR
IS THIS JUST GOING TO BE
BURDENSOME WITH NO REAL BENEFIT
TO THE UNDERSTANDING OF THE
PATIENT JOURNEY OR THE PATIENT
NEEDS INTO PROGRESSION SO I
THINK WE HAD A LOT OF OPEN
CONVERSATION WHICH I REALLY,
REALLY APPRECIATED. WE WERE
ABLE TO CHANGE OUR SCHEDULE
QUITE A BIT FROM TIME TO TIME SO
IT WASN'T AS BURDENSOME TO THE
PATIENTS AND FOR ME BEING THE
ADULT AND THE ONE WHO HAS LIVED
WITH THIS DISEASE THE LONGEST I
AM THE EXPERT IN THE ROOM AND I
THINK THAT IS SO IMPORTANT TO
UNDERSTAND AS RESEARCHERS, YOUR
PATIENT IS THE EXPERT. SO YOU
MAY HAVE ALL THE SCIENTIFIC
INFORMATION BUT REALLY LIVING
THE DISEASE IS SOMETHING SO
DIFFERENT. I CAN TELL YOU FROM
MY EXPERIENCE, YOU KNOW, WHAT
PARTS OF MY BODY MY DISEASE HAS
IMPACTED WITHOUT YOU RUNNING TEN
BATTERY OF TESTS, RIGHT? SO I
THINK IT'S A GIVE AND TAKE.
IT'S ABOUT UNDERSTANDING TO BE
ABLE TO THEN SCHEDULE
ASSESSMENTS ACCORDINGLY AND
REALLY TAKE THAT INPUT SO THAT
WAS REALLY VERY CRUCIAL FOR OUR
JOURNEY AND OUR PATH AT THE
CENTER AND I HAVE TO SAY ALL OUR
RESEARCHERS WHO WERE SO OPEN AND
SO WILLING TO UNDERSTAND THAT
AND PIVOT AS NEEDED. I THINK
THAT AS ALREADY MENTIONED, IT'S
THE RELATIONSHIP BETWEEN THE
RESEARCHERS THAT CAN BE SO
EMPOWERING. AND POSITIVE AND
MAKE THE EXPERIENCE MORE
MANAGEABLE. WE HAVE HAD A
REALLY POSITIVE EXPERIENCE. I
WOULD SAY THAT, YOU KNOW, IT'S
IMPORTANT FOR DISEASES LIKE
OURS. LIKE SOMETIMES WE COME TO
THE CENTER I HAD NOTICED THAT
THE PERSON WHO DID MY EYE TEST
OR THE ONE WHO DID MY AUDITORY
TEST WAS NOT THE SECOND PERSON
THE SAME TIME AROUND SO IT
ALMOST FEELS LIKE THEY DIDN'T
UNDERSTAND WHAT THEY WERE SEEING
AND SO THEY STARTED ORDERING
MORE THINGS BUT IF WE HAD THE
SAME PERSON WHO WAS DOING THE
TESTING AT THE SAME SET OF
SCHEDULE, THEY WOULD HAVE BEEN
ABLE TO PIECE THE -- MAKE THE
PIECES MAKE SENSE IN THE PUZZLE.
AND THEY WOULDN'T THEN, YOU
KNOW, KEEP US LONGER OR, YOU
KNOW, SAY, OH I THINK I FOUND
SOMETHING AND THAT CAUSES A LOT
OF ANXIETY FOR US AS PATIENTS,
RIGHT? SO THAT WAS A CHANGE
THAT I HAD REQUESTED THAT, YOU
KNOW, EVERY PERSON THAT DOES OUR
TESTS REMAIN THE SAME AS MUCH AS
POSSIBLE. AND SO THEN THE STUDY
TEAM BEGAN TO SEE THE PICTURE.
RIGHT? IT WAS THE STORY STARTED
MAKING SENSE AND I THINK
RESEARCH IS ABOUT THAT.
ESPECIALLY IN NATURAL STUDY IT'S
ABOUT FINDING THE PUZZLE PIECES
THAT THEN TELL A STORY OF THE
DISEASE. BUT IF YOU KEEP
CHANGING PEOPLE OUT. THEN THAT
BECOMES HARDER AND MORE
BURDENSOME ON THE PATIENT TO
HAVE TO TELL, YOU KNOW, MY STORY
FROM A-Z AGAIN TO A PERSON
SOMETIMES THAT BECOMES REALLY
HARD AND YOU LOSE IMPORTANT
PIECES OF THE STORY. SO THAT
WAS THE FEEDBACK I HAD GIVEN TO
MY RESEARCH TEAM AND I QUICKLY
MADE THAT CHANGE AND THEY TRIED
TO GET EVERYBODY ON BOARD THAT
WAS SEEING THE DIFFERENT
PATIENTS IN OUR COMMUNITY TO BE
THE SAME BECAUSE REMEMBER ONCE
WE ENTERED THE NATURAL HISTORY
STUDY WAS NOT JUST ME AND MY
CHILDREN BUT WE HAD THREE OTHER
PATIENTS THAT CAME FROM
DIFFERENT PARTS OF THE WORLD AND
THIS DISEASE JOURNEY, THAT ONE
PERSON WAS SEEING ALL THESE
CHILDREN. AND NOT LIKE FOUR
DIFFERENT PEOPLE SEEING, YOU
KNOW, THESE CHILDREN, YOU JUST
CAN'T MAKE SENSE OF THE STORY.
SO THAT WAS SOMETHING THAT WAS
IMPORTANT TO US. THE OTHER
THING I WOULD SAY IS THE
CHILDREN. SO MY KIDS ARE RIGHT
NOW 14 AND 16 AND WHEN THEY
STARTED, THEY WERE YOUNGER THAN
THAT. TWO YEARS YOUNGER THAN
THAT. IT WAS REALLY DIFFICULT
IN THE BEGINNING. THERE IS A
SENSE OF LIKE ONCE YOU ENTER,
ESPECIALLY SINCE AFTER COVID,
SOME OF THE RULES HAD CHANGED SO
THERE WAS A SENSE OF FEELING
TRAPPED. I KEPT HEARING FROM MY
KIDS THAT THEY WERE GETTING
RESTLESS AND FRUSTRATED AND I
THINK WHILE THE CENTER HAS LOTS
OF THINGS FOR CHILDREN WHO ARE
YOUNG
YOUNGER I THINK THAT TEENAGE OF
13 AND ABOVE WAS REALLY HARD.
THERE'S NOT AGE APPROPRIATE
THINGS TO DO. AND WE WEREN'T
ABLE TO LEAVE THE CENTER. WHICH
I FOUGHT FOR. BUT I LOST. AND
JUST KEPT SAYING IT WAS
IMPORTANT FOR THESE CHILDREN TO
HAVE PATH TO LEAVE THE CENTER IF
THEY'RE NOT HOOKED ONTO ANYTHING
THEY SHOULD BE ABLE TO LEAVE THE
CENTER BUT WE WEREN'T ABLE TO
AND THAT FEELING OF BEING
TRAPPED IN THERE CAN INCREASE A
SENSE OF FEELING, YOU KNOW,
STRESS AND ANXIETY. EVEN THOUGH
THEY KNOW WHAT THEY'RE THERE
FOR, THEY CAN UNDERSTAND IT.
THEY WERE FINDING IT HARD TO
COPE WITH THE WEEK LONG STAY
ESPECIALLY WITH THE SENSE OF I
FEEL TRAPPED AND HELPLESS
COMPOUNDED WITH THE FACT THAT
CHILDREN VALUE AUTONOMY, THE
ABILITY TO MAKE CHOICES AND
BEING IN A CONTROLLED
ENVIRONMENT CAN BE
DISHEARTENING, IT'S NOTHING THE
FAULT OF OUR RESEARCH TEAM IT'S
JUST THE WAY RESEARCH IS SET UP.
SO I'M WONDERING HOW TO MAKE
THAT EASIER TO MAKE IT TO LEAVE
THE CENTER. THERE'S LOTS OF
THINGS AROUND TO DO IN THE AREA.
WE DIDN'T HAVE A WAY TO DO THAT.
THERE WAS NO PATH TO GET ON A
BUS AND GO SOMEWHERE SO BRINGING
IN SOME OF THOSE WOULD BE NICE.
THE OTHER THING IS THAT YOU
KNOW, FOR A WEEK, YOU ARE
WITHOUT YOUR FRIENDS, YOU'RE
DISCONNECT FROM SCHOOL. SO
THERE IS A LOT OF ANXIETY ON HOW
TO GET BACK TO OUR -- THE
ACADEMIC STANDING OR THE MISSING
OUT OF ACADEMICS SO HOW CAN WE
FACTOR THAT IN ESPECIALLY WHEN
YOUR WHOLE DAY IS BUSY WITH
ACTIVITY AND TESTS AND THINGS.
I THINK IT DOES TAKE A CHUNK OUT
OF THEIR SOCIAL LIVES. SO, YOU
KNOW, THERE WAS, ESPECIALLY FOR
US, THERE'S NO PEER GROUP,
RIGHT? THERE'S NO PEER GROUP
ACTIVITY BECAUSE NOBODY ELSE HAS
THIS DISEASE. SO THAT IS
ANOTHER THING TO CONSIDER. TO
GET CHILDREN IN THESE
DISCUSSIONS AND SORT OF VOICE
IT. MORE AND MORE WHEN I THINK
ABOUT IT, THE MENTAL HEALTH
IMPACT OF BEING IN A STUDY OR
BEING IN RESEARCH IS NOT GIVEN
ENOUGH ATTENTION. AND SO, YOU
KNOW, ARE THERE COUNSELORS THAT
THESE KIDS CAN TALK TO? AS THEY
ARE GOING THROUGH ALL THIS?
BUT, YES, I WILL STOP THERE
BECAUSE....
>> THANK YOU VERY MUCH THAT WAS
LOVELY AND A LOT TO TAKE IN.
AND I AM GOING TO PUT A COUPLE
WORDS IN YOUR MOUTH. IN
TRANSITIONING IS WHAT IS THE
MOST IMPORTANT THING FOR STUDY
MEMBERS TO KNOW ABOUT
PARTICIPANTS AND ONE YOU
HIGHLIGHTED IS THAT YOU'RE THE
EXPERT. ESPECIALLY WHEN IT'S A
RARE DISEASE, THAT YOU'RE A TEAM
MEMBER. AND THAT YOU WANT, LIKE
YOU WANT TO BE A TEAM MEMBER.
THAT MAY NOT BE TRUE FOR
EVERYONE BUT FOR YOU, IN
PARTICULAR, OF BEING A TEAM
MEMBER AND RESPECTING THAT ROLE
AND THEN ALSO JUST THAT
PARTICIPANTS ARE HUMANS. RIGHT?
NOT TO SAY THAT THAT'S, YOU
KNOW, OF COURSE THAT'S OBVIOUS.
BUT THE SORT OF THINGS THAT YOU
WERE TALKING ABOUT IN TERMS OF,
YOU KNOW, YOUR KIDS, THAT THEY
ARE HUMAN TEENAGERS. AND HAVE
PARTICULAR, YOU KNOW, NEEDS AND
INTERESTS THAT IT WOULD BE
IMPORTANT FOR THE STUDY TEAM TO
TAKE, YOU KNOW, TO KEEP IN MIND.
ANYTHING ELSE YOU WANT TO ADD TO
THAT LIST OF WHAT YOU'VE MADE
YOUR STUDY TEAM AWARE OF OR
ANYTHING IN PARTICULAR THAT YOU
WANT TO EMPHASIZE ABOUT WHAT
THEY SHOULD KNOW ABOUT THEIR
PARTICIPANTS?
>> YES, I WILL GIVE YOU AN
ILLUSTRATIVE EXAMPLE AFTER A
REALLY LONG DAY OF TESTS AND
THIS WAS PROBABLY BY THE FOURTH
DAY, OF THE WEEK, WE WERE THERE,
I OVERHEARD A CONVERSATION
BETWEEN MY KIDS AND MY YOUNGER
ONE, HE WAS REALLY TIRED. YOU
KNOW, THEY HAD POKED HIM MANY
TIMES AND MISSED THE VEIN AND HE
WAS REALLY EMOTIONALLY KIND OF
SPENT AND WE HAD GONE INTO MANY
EASIER TESTS, ONE THAT HE
ACTUALLY ENJOYED DOING AND HE
LOOKED AT HIS BROTHER AND SAID
DO WE HAVE TO DO THIS AND MY
OLDER SON SAID TO HIM, WE DON'T
HAVE A CHOICE. IT'S FOR THE
GREATER GOOD. RIGHT? AND SO MY
HEART AS A MOTHER REALLY BROKE
BECAUSE I WAS THINKING ALL THIS
TIME I WAS THINKING THAT, YOU
KNOW, AND WE HAD SEVERAL
DISCUSSIONS ABOUT IT. WE TALKED
ABOUT IT BEFORE WE REACHED CARE
AND AS A MOM TO HEAR THAT AND
I'M GETTING EMOTIONAL JUST
THINKING ABOUT IT BUT AS A MOM
THINKING ABOUT IT DO WE CLEARLY
EXPLAIN TO THE CHILDREN THAT
THEY HAVE A CHOICE. YOU KNOW,
BEING THE FIRST IS DRAMATIC.
IT'S COOL, IT'S EXCITING. BUT
DO WE HAVE A CHOICE IN THE
MATTER? ARE WE MAKING IT CLEAR
TO THE CHILDREN IN LANGUAGE THAT
IS CLEARLY UNDERSTANDABLE THAT
THEY HAVE A CHOICE TO DO THIS?
BECAUSE SOMETIMES A CHOICE IS
TAKEN OUT OF OUR HANDS BECAUSE
THEY KNOW IF THEY DON'T DO THIS
THEY DON'T GET TREATMENT.
THEY'LL CONTINUE TO SORT OF LIVE
WITH JANSEN'S IN A VERY
DEBILITATING WAY. BOTH MY KIDS
USE WHEELCHAIRS THE FACT THAT
THIS IS A VERY IMPORTANT
DISCUSSION. IN THAT HOW DO WE
COMMUNICATE WITH THE CHILDREN
AND IN WAYS THAT HAVE R
INDIVIDUALIZED. YOU'RE NOT
TALKING TO CHILDREN.
THEY'RE NOT ALL THE SAME. EVEN
BETWEEN MY TWO KIDS THEY HAVE
DIFFERENT APPROACH TO THE TRIAL.
SO THAT'S SIGNIFICANT TO
UNDERSTAND THAT EVERYONE GOING
INTO THE TRIAL IS AN INDIVIDUAL.
THEY HAVE THEIR OWN FEARS, THEIR
OWN UNDERSTANDING OF THE PROCESS
AND THEIR OWN FEELINGS OF HOW
THEY'RE DEALING WITH IT. WHICH
IS WHY I'M SAYING IT IS TO
IMPORTANT TO HAVE A COUNSELOR ON
BOARD. WHEN YOU ARE DEALING
WITH A CHILDREN, THAT'S SO
CRITICAL TO MAKE THEM
UNDERSTAND, TO ALLOW THEM TO
VOICE THEIR OPINIONS. SHARE
THEIR CONCERNS IN WAYS THAT ARE
COGNITIVELY RELEVANT TO THAT
COMMUNITY AS WELL AS THE
CULTURAL SIGNIFICANCE OF, OR --
WITH THE CULTURAL SENSITIVITY
THAT THEY'RE BRINGING TO THAT
RESEARCH. RIGHT? SO ALL OF
THESE BECOME SO IMPORTANT TO THE
DISCUSSION. IT'S NOT YEAH,
HERE'S YOUR NEXT TEST, NO, WHAT
WAS THE DISCUSSION BEFORE THAT?
WHAT ARE THE -- IT'S NOT JUST
WHAT ARE YOU GOING TO EXPECT TO
HAVE TO DO BUT TALK TO THEIR
MIND? TALK TO THEIR HEART?
TALK TO THEIR SOUL. SO I THINK
THIS IS SOME OF THE CHALLENGES I
THINK WE NEED TO BEGIN TO
UNPACK.
AND, YOU KNOW, I KEEP REFLECTING
THAT AS A CHILD WHEN I WENT INTO
THE HOSPITAL, I FELT LIKE I WAS
SO NAIVE AND SO DUMB AND
CHILDREN TODAY ARE NOT. THEY
ASK QUESTIONS, THEY WANT TO
KNOW. THEY WANT TO BE INVOLVED.
THEY WANT TO FEEL THAT THEY HAVE
SOME CONTROL. YOU KNOW, IF I
WAS SEVEN AND THEY TOLD ME TO
GET UP ONTO THIS YOU KNOW STUDY
TABLE I WOULD HAVE JUST JUMPED
UP WITHOUT ANY THOUGHT BUT
TODAY'S CHILDREN ARE NOT LIKE
THAT AND I THINK WE HAVE TO
THINK ABOUT THAT AND THINK ABOUT
HOW ARE WE CHANGING OUR STUDY
ENVIRONMENT TO REFLECT THESE
CHANGING NORMS.
>> GREAT. NOW NOT TO PUT YOU ON
THE SPOT, LIAM AS THE YOUNGEST
MEMBER OF OUR PANEL, BUT I'M
WONDERING IF YOU HAVE HAD ANY
SORT OF THOUGHTS AS NINA'S BEEN
TALKING ABOUT, YOU KNOW, WERE
THERE OPPORTUNITIES FOR YOU TO
TALK WITH FOLKS ABOUT YOUR
PARTICIPATION OR DID YOU FEEL
SORT OF ABLE TO MAKE A DECISION.
TELL US A LITTLE MORE ABOUT THAT
IF YOU DON'T MIND.
>> YEAH, SO, I MEAN, I THINK THE
BEST WAY TO EXPLAIN THAT IS I
DID HAVE QUESTIONS LIKE SHE
MENTIONED. BUT IT WAS BEFORE
THE CLINICAL TRIAL. I WAS
LOOKING AT ALL MY OPTIONS KIND
OF SITUATION. LIKE THIS --
THERE WAS ANOTHER TRIAL THAT WAS
KIND OF THE SAME -- IT'S LIKE
THE COMPETITIVE MY TRIAL THAT I
COULD HAVE DONE. I COULD HAVE
DONE GENE THERAPY OR I COULD
HAVE DONE A HYDROCORTISONE
RELEASE DRUG. BASICALLY I HAD
E-MAILED SO MANY DOCTORS ABOUT
THESE OTHER TRIALS BECAUSE I I
WANTED TO MAKE SURE THAT I
DISE --
WANTED TO MAKE SURE I WASN'T
GETTING A BIASED ANSWER EITHER.
I DIDN'T ASK THE TRIAL DOCTORS,
AS IN THE PEOPLE DOING THE
TRIALS, I WOULD ASK DOCTORS THE
ENDOCR
ENDOCRINOLOGISTS ABOUT THE
TRIALS BUT WERE NOT PART OF THE
TRIALS. I E-MAILED A DOCTOR,
YOU KNOW, WHAT DO YOU THINK OF
THE GENE THERAPY TRIAL? AND HE
SAID, PERSONALLY, I WOULD NOT DO
IT, I WOULD NOT RECOMMEND IT
AND, YOU KNOW, HE'S RIGHT
BECAUSE I WOULDN'T SAY HE'S
RIGHT BUT TWO YEARS LATER THE
GENE THERAPY TRIAL GOT SHUT DOWN
BECAUSE THE MEDICAL TEAM DECIDED
THAT THEY DIDN'T WANT TO PURSUE
IT ANYMORE THAT THERE WASN'T
ENOUGH EVIDENCE OF, YOU KNOW,
IMPROVEMENT WITH THE GENE
THERAPY SO THERE WAS JUST NO
POINT. AND IF I HAD JOINED THAT
GENE THERAPY WELL I COULDN'T
HAVE JOINED THIS TRIAL WHICH,
YOU KNOW, HAS MORE PROMISE
CLEARLY BECAUSE NOW IT'S
ACTUALLY ABOUT TO BE RELEASED
VERSUS THE GENE THERAPY WHICH
AGAIN IS SHUT DOWN. AND, BUT,
YOU KNOW, OVERALL I WOULD SAY
LIKE WITH MY EXPERIENCE OF
PARTICIPATING, ONCE I GOT IN THE
CLINICAL TRIAL IT WAS PRETTY
MUCH, YOU KNOW, NORMAL. I FELT
COMFORTABLE. THE ONLY PROBLEMS
I WOULD SAY WOULD BE THAT AS,
LIKE, STUDY TEAM MEMBERS WOULD
NEED TO KNOW ABOUT IS ONE, BE A
BIT MORE, LIKE, I GUESS CARING
IN THE SENSE THAT SOME OF THE
PROCEDURES CAN BE EXTREMELY
INVASIVE. AND YOU GUYS, SOME OF
THEM CAN BE LIKE WHEN DO YOU
WANT TO SCHEDULE IT? I'M LIKE
LET ME THINK ABOUT THAT REAL
QUICK AND THE OTHER ONE WOULD
BE, YOU KNOW, I GUESS IT COMES
DOWN TO THE FACT THAT SCHEDULING
AT THE END OF THE DAY, I GET
THAT THE CLINICAL TRIAL PEOPLE
ALSO HAVE THEIR OWN SCHEDULES.
BUT THERE CAN BE TIMES WHERE IT
BE LIKE OH CAN YOU COME IN
MONDAY, CAN YOU COME IN TUESDAY
BUT I HAD JUST COME IN MONDAY
LIKE FIVE TIMES IN A ROW AND I
CANNOT MISS ANOTHER MONDAY
CLASS. BUT FOR THEM THAT'S WHAT
THEIR SCHEDULE WORKS SO IT KIND
OF BECAME LIKE A, YOU KNOW, A
SITUATION WHERE WE HAD TO FIGURE
OUT HOW TO BALANCE EACH OTHER'S
SCHEDULE, NOT JUST, YOU KNOW,
NOT JUST MY SCHEDULE AT THE END
OF THE DAY OR THEIR SCHEDULE
BECAUSE, IF WE HAVE STUFF WE
HAVE STUFF AND WE CAN'T JUST
DROP IT ALL. BUT, YEAH.
>> GREAT. THANK YOU. HOW ABOUT
YOU IN TERMS OF YOU SHARING YOUR
THOUGHTS ABOUT THE STUDY TEAM S
THERE ANYTHING YOU HAVEN'T
MENTIONED THAT YOU WANT STUDY
TEAMS TO KNOW ABOUT YOU OR
PARTICIPANTS MORE GENERALLY?
>> SURE, ABSOLUTELY.
I AM GOING TO ECHO KIND OF WHAT
NINA WAS SAYING FOR
COMMUNICATION AND LIAM FOR
CARING I WILL ADD ACCOMMODATING.
A LITTLE BIT GOES A LONG WAY I
CAN GIVE YOU GUYS AN EXAMPLE.
EVERY PATIENT IS VERY UNIQUE AND
FOR ME ONCE MY EYES GOT DILATED
MY VISUAL ACUITY WAS VERY
MINIMAL AND MY TECHNICIAN I WAS
TELLING YOU GUYS ABOUT, HE WAS
SO GOOD AT ONCE THOSE EYES WERE
GETTING SUPER DIE LATED AND WE
WERE GOING TO GO TO ANOTHER
TESTING HE WAS SO GREAT AT
BECOMING MORE VOCAL AND VERBAL
WITH THINGS.
AND HE SAID HEY, I'M GOING TO
WALK MY HAND RIGHT NOW AND AN
ALCOHOL WIPE TO WIPE DOWN THE
MACHINE THAT YOU'RE GOING TO BE
USING. THOSE LITTLE SMALL
THINGS MADE ME FEEL SO
COMFORTABLE AND SAFE BECAUSE I
WAS -- I CAN'T SEE, I DON'T KNOW
WHAT IS GOING ON. SO I KNOW FOR
YOU GUYS AS STUDY MEMBERS EVERY
PATIENT ROOM THERE'S A LITTLE
SMALL CHANGE OR TWEAK I THINK
WILL MAKE A HUGE DIFFERENCE FOR
YOU GUYS, COMMUNICATION, YES,
ABSOLUTELY BUT KIND OF RINGING
THAT ACCOMMODATION, EVEN JUST
ASKING THE PATIENT HOW CAN I
MAKE THIS EXPERIENCE A LITTLE
BIT MORE EASIER? BECAUSE IT IS
LONG DAYS AND A LOT OF TIMES I
KNOW. JUST WANT TO GET ALL
THOSE TEN TESTS DONE BUT THOSE
SMALL LITTLE THINGS MAKE A HUGE
DIFFERENCE AND I CAN'T EMPHASIZE
THAT MORE FOR MAKING THE
EXPERIENCE BETTER FOR THE
PATIENT.
>> AND HOW ABOUT YOU, ELLEN?
>> I AGREE, EVEN THOUGH WHEN I
GO INTO PHLEBOTOMY, THEY REALLY
DIDN'T WANT TO DO ME. THEY
WOULD. YOU KNOW. AND THEN
THEY'D -- WE'D ALL CELEBRATE
WHEN THEY FINALLY GOT A VEIN
LIKE OH, YAY, WE GOT ONE. SO
THEY FELT MY ANXIETY THAT, OH,
IS THIS PERSON GOING TO BE ABLE
TO GET IT SO NOW I ALWAYS HAVE
TO ASK FOR THEIR BEST PERSON AND
A HEATING PAD. I KNOW WHAT TO
DO NOW. AND THEY KNOW WHAT TO
DO. SO IT'S ALL WORKED OUT
BECAUSE AGAIN THEY TREAT YOU AS
A PERSON. YOU KNOW, AND I THINK
THAT'S WHAT EVERYBODY'S HAD TO
SAY, THEY TREAT YOU AS AN EA
EARN -- PERSON.
>> ONE MORE QUESTION FROM ME AND
THEN WE'LL OPEN IT UP TO THE
AUDIENCE AND I ALSO WANT TO
WELCOME TONY JONES WHO JOINED
US. SHE'S MOST RECENTLY THE
PARTICIPANT ADVOCATE HERE AT THE
CLINICAL CENTER AND HAS MOVED
ONTO A DIFFERENT POSITION BUT
WOULD LOVE TO HEAR ANY THOUGHTS
YOU MIGHT WANT TO ADD TONY, ONCE
WE FINISH UP. SO THE LAST
QUESTION I WANTED TO ASK AND
THEN START WITH YOU ELLEN ABOUT
IF I AM THINKING ABOUT COMING TO
THE CLINICAL CENTER OR ENROLLING
IN A CLINICAL TRIAL, WHAT SORT
OF ADVICE WOULD YOU HAVE FOR ME
IN MAKING SURE THAT I KNOW WHAT
I AM DOING OR WHAT TO EXPECT?
>> I WOULD FIRST WANT TO KNOW
WHY. WHY ARE YOU INTERESTED IN
THIS TRIAL? AND WHAT ARE YOUR
HOPES FOR THE TRIAL? BECAUSE A
HOPE SHOULD NOT BE A CURE.
BECAUSE YOU'RE NOT DOING IT FOR
A CURE. THAT'S NOT GOING TO
HAPPEN. USUALLY BUT TO FIND OUT
MORE PERMANSONALLY FROM YOU AS
PERSON WHY IS IT THAT YOU WANT
TO DO THIS AND THEN I WOULD OF
COURSE TELL YOU WHAT A SUCCESS I
HAD. BUT, AND KIND OF THINGS
THAT I'VE DONE AND WHAT, YOU
KNOW, THERE'S CONSTRUCTION ALL
THE TIME. SO YOU HAVE TO GET
AROUND THAT. FINDING A WAY
AROUND. AND LET THEM KNOW
THAT -- AND I DID REFER ANOTHER
PERSON THAT WAS COMING FOR A
TRIAL AND I SAID I WILL GO WITH
YOU. THE FIRST TIME. JUST SO
YOU CAN SEE WHERE PHLEBOTOMY IS
AND HOW TO GET TO THE PLACES AND
SHE WAS VERY GRATEFUL FOR THAT
AND SHE'S STILL PART OF A TRIAL.
SO....
>> I REALLY APPRECIATE WHERE YOU
STARTED. RIGHT? OF, YOU KNOW,
WANTING TO KNOW WHERE THEY'RE
COMING FROM FIRST. BEFORE SORT
OF SAYING, OH, APPRECIATE THAT.
>> I WOULD LOVE TO START WITH
JUST ME. BECAUSE IT'S ALWAYS
ABOUT ME. I WILL GIVE THEM A
CHANCE FIRST.
>> GOOD. GOOD.
>> LET'S SEE, I WOULD PROBABLY
SAY THE FIRST THING, DON'T BE
SCARED. I WAS -- IT'S REALLY
OVERWHELMING HERE. THERE'S A LOT
OF INFORMATION THEY GIVE AT YOU
AT THE SAME TIME AND JUST BE
WILLING TO NAVIGATE WITH THEM I
THINK DON'T BE SCARED, TAKE THAT
FIRST STEP AND I WISH I HAD THIS
WHEN I FIRST STARTED, HAVING A
BUDDY OR SOMEONE WHO HAS BEEN
HERE, NOW THE CLINICAL WEBSITE
HAS THIS GREAT WEB PAGE OF
PATIENTS TALKING ABOUT THEIR
EXPERIENCE, REACHING OUT TO THEM
AND SAYING, HEY, I'M THINKING
ABOUT DOING THIS, WHAT IS YOUR
EXPERIENCE. I WISH I HAD THAT.
I WAS SO IN THIS BOX AND I WAS
LIKE OKAY I GUESS I WILL DO IT.
HAVING CREATE A NETWORK WHETHER
IT'S IN THE CLINICAL CENTER OR
OUTSIDE HAVING THAT SUPPORT
SYSTEM BECAUSE IT IS A HUGE
DIFFERENCE. IT IS OVERWHELMING
COMING HERE AND I AM SURE YOU
GUYS COMING TO THIS AUDITORIUM
MIGHT HAVE BEEN HARD. SO JUST,
YEAH, NOT BEING SCARED AND JUST
FINDING THAT NETWORK EVEN IF
IT'S ONE PERSON OR A BUNCH OF
PEOPLE I THINK THAT'S A HUGE
THING THAT IS GOING TO HELP THE
PATIENT NAVIGATE THIS.
>> GREAT. HOW ABOUT YOU, LIAM?
>> I WOULD JUST SAY FOR ME I
HAVE A UNIQUE PERSPECTIVE I FEEL
BUT LOOK AT ALL THE ADVANTAGES
OF PARTICIPATING IN RESEARCH, ET
CETERA. THAT YOU CAN TAKE
ADVANTAGE OF BECAUSE FOR ME,
BEFORE JOINING THIS CLINICAL
TRIAL I DIDN'T THINK I WAS GOING
TO BE YOU KNOW ANYTHING MEDICAL
RELATED OR HAVE ANY INTEREST IN
MEDICAL STUFF AT ALL BUT ONCE I
JOINED THE MEDICAL TRIAL, LIKE
OUTSIDE OF THE MIEDICAL TRIAL I
WAS ABLE TO TAKE ADVANTAGE OF
OTHER OPPORTUNITIES BECAUSE I
WAS IN THIS MEDICAL TRIAL LIKE
FOR INSTANCE I WAS ABLE TO JOIN
THE FDA PANEL ON HOW, YOU KNOW,
CLINICAL TRIAL INITIATIVES THAT
THEIR PARTNERSHIP WITH DUKE. SO
I WORK WITH THEM NOW AND, YOU
KNOW, AS A COLLEGE STUDENT
THAT'S BENEFICIAL FOR ME IN
TERMS OF A RESUME KIND OF
SITUATION. AND I ALSO GET TO
WORK WITH THE CLINICAL TRIAL
COMPANY THAT I'M IN NOW AS AN
ADVISOR AS WELL, A PATIENT
ADVISOR, BEFORE THEY RELEASE THE
DRUG SO THEY CAN GET ADVICE ON
HOW TO GO ABOUT IT FROM A
PATIENT PERSPECTIVE AND OVERALL,
I MEAN, YOU KNOW, IF I HAD NOT
JOINED THE CLINICAL TRIAL I
WOULDN'T HAVE HAD THOSE
OPPORTUNITIES AND I WOULDN'T
HAVE HAD THIS OPPORTUNITY AS
WELL. IT'S DEFINITELY LIKE
EXTERNAL OPPORTUNITIES OUT THERE
THAT YOU WOULDN'T REALLY THINK
OF WHEN YOU'RE CONSIDERING
PARTICIPATING IN THE RESEARCH
BUT LIKE ONCE YOU START GETTING
INTO THE RESEARCH AND YOU START
BECOMING MORE INVOLVED BECAUSE
HERE YOU ARE GETTING TESTED AS A
PERSON YOU TOOK THAT BIG STEP
THAT ISN'T KNOWN ABOUT THAT CAN
BE RISKY. YOU BECOME MORE
CARING ABOUT, YOU KNOW, YOUR
CONDITION ABOUT THE DRUG AND
JUST OVERALL A COMMUNITY THAT
YOU'RE INVOLVED IN SO, I MEAN,
YEAH, MY BIGGEST ADVICE IS LOOK
AT ALL YOUR OPTIONS AND
CONSIDER, YOU KNOW, THE
POTENTIAL ADVANTAGES OF JOINING,
YOU KNOW, A CLINICAL TRIAL.
>> GREAT, THANK YOU AND I WILL
GIVE YOU THE LAST WORD NINA
ABOUT WHAT YOU WOULD SHARE IF I
WAS THINKING ABOUT ENROLLING?
>> THANK YOU. YEAH. SO BECAUSE
I'VE GONE THROUGH THIS
EXPERIENCE AND BECAUSE I'M ALSO
THE LEAD ON -- THE LEADER IN MY
COMMUNITY AND I AM THE DIRECTOR
OF OUR FOUNDATION AND I HAVE TO
ENCOURAGE OTHERS IN MY COMMUNITY
TO PARTICIPATE, I REALLY THOUGHT
LONG AND HARD ABOUT THIS
QUESTION. AND ACTUALLY I
DEVELOPED A SORT OF CHEAT SHEET.
THE FIRST THING I ALWAYS SAY IS
DO YOUR RESEARCH AND I WOULD
DEFINITELY SHARE INFORMATION
ABILITY FAMILIARIZING YOURSELF
WITH WHAT IS THE PURPOSE OF THE
STUDY, WHAT ARE THE PROCEDURES,
NOW THAT WE HAVE THAT CLEARLY
DEFINED, HERE IS WHAT THAT LOOKS
LIKE, WHAT IS THE POTENTIAL
RISKS AND BENEFITS OF THIS
RESEARCH? AND ASK QUESTIONS
UNTIL YOU FEEL COMFORTABLE WITH
THE INFORMATION. AND IT'S
REALLY IMPORTANT TO HAVE THAT
SPACE TO ASK THOSE QUESTIONS.
COMMUNICATE OPENLY. TALK TO THE
RESEARCH TEAM. DON'T HESITATE
TO DISCUSS CONCERNS OR ASK FOR
ANY CLARIFICATION. OPEN
COMMUNICATION LIKE I'VE SAID,
OVER AND OVER IN THIS DISCUSSION
IT HELP TO BUILD THAT TRUST THAT
IS SO IMPORTANT. CONSIDER YOUR
OWN COMFORT LEVEL. LIKE YOU
HAVE TO ASSESS YOUR READINESS.
YOU THINK ABOUT WHETHER YOU FEEL
EMOTIONALLY AND PHYSICALLY
PREPARED TO PARTICIPATE. I
MEAN, SOME OF THESE STUDIES,
IT'S A LONG HAUL AND IF
SOMETHING DOESN'T FEEL RIGHT IF
YOU DON'T FEEL COMFORTABLE WITH
ONE PERSON ON YOUR STUDY TEAM,
IT'S OKAY TO DECLINE OR TO WAIT
UNTIL YOU FEEL MORE COMFORTABLE.
I THINK THAT'S REALLY IMPORTANT.
I NEVER UNDERSTOOD HOW MUCH
OF -- HOW MUCH THIS IS GOING TO
TAKE OUT OF ME AND MY COMMUNITY
TO BE INVOLVED AND YET KNOW THIS
IS THE ONLY WAY WE WOULD GET TO
A TREATMENT. IT'S A SIGNIFICANT
STEP AND SOMETIMES YOU FEEL LIKE
YOU HAVE TO CHOICES. AND SO
WHEN YOU'RE IN THAT SITUATION,
YOU REALLY NEED TO ASSESS ALL OF
THESE POINTS. INVOLVE YOUR
SUPPORT SYSTEM, ENGAGE WITH YOUR
FAMILY AND FRIENDS, DISCUSS YOUR
DECISION WITH YOUR LOVED ONES.
YOU KNOW, THEIR SUPPORT CAN BE
SO IMPORTANT TO THE WHOLE
PROCESS AND HOW YOU NAVIGATE
THIS EXPERIENCE. AND ANOTHER
IMPORTANT THING IS TO KNOW YOUR
RIGHTS. UNDERSTAND INFORMED
CONSENT. FAMILIARIZE YOURSELF
WITH YOUR RIGHTS AS A
PARTICIPANT. INCLUDING YOUR
RIGHT TO WITHDRAW FROM THE STUDY
WITHOUT ANY ADVERSE, YOU KNOW,
EFFECT OR ANY KIND OF PENALTY.
YOU KNOW, IN DISEASES, RARE
DISEASES, WE HAVE HAD PATIENTS
TELL ME WE DON'T WANT TO
PARTICIPATE. RIGHT? BECAUSE
THEY DON'T TRUST THE SYSTEM OR
THEY DON'T TRUST. ESPECIALLY,
AND THIS IS SO IMPORTANT TO
KNOW, WE HAVE HAD PATIENTS FROM
OUTSIDE THE U.S. WHO DID NOT
WANT TO PARTICIPATE BECAUSE THEY
HAVE A VERY NEGATIVE IMPRESSION
OF AMERICAN MEDICINE OR THE NIH
AND THAT IS IMPORTANT TO KNOW.
AND THAT IS IMPORTANT TO COUNTER
AND TO MAKE OUR COMMUNITY
UNDERSTAND SO THE TRANSPARENCY
OF WHAT WE'RE DOING IS SO
IMPORTANT. BE AWARE OF TIME
COMMITMENTS LIKE PLAN FOR WHAT
THESE APPOINTMENTS ARE GOING TO
LOOK LIKE. WE HAVE TO BE REALLY
REALISTIC ABOUT THE TIME THAT'S
REQUIRED FOR THESE VISITS,
TESTS, THE FOLLOW UPS AND ALL OF
THE UNKNOWNS THAT COME WITH IT.
SO EXPECT THE UNEXPECTED.
RIGHT? WE HAVE TO STAY FLEXIBLE
IN DOING THAT. BE OPEN TO
SHARING WHAT'S UNEXPECTED AND
THE OUTCOMES THAT COME WITH IT
AND BE ABLE TO ADAPT AND TALK TO
YOUR TEAM AND MANAGE ANY OF THE
SURPRISES THAT MIGHT HAPPEN
ALONG THIS JOURNEY. AND IF AT
ALL POSSIBLE, IF THERE IS
OPPORTUNITY TO CONNECT WITH
OTHER PARTICIPANTS I THINK
THAT'S IMPORTANT TO SEEK
COMMUNITY SUPPORT AND THEN
FINALLY I WOULD SAY TRUST YOUR
INSTINCTS, RIGHT? FOLLOW YOUR
GUT, ULTIMATELY TRUST YOUR
INSTINCTS ABOUT O WHETHER TO
PARTICIPATE.
THE REALITY IS IT'S A HARD ROAD
TO BE THE FIRST. AND WE HAVE TO
BE PREPARED MENTALLY FOR IT.
IF IT'S NOT YOUR ROAD IF IT'S
NOT SOMETHING YOU FEEL GOOD
ABOUT IT'S OKAY TO STEP BACK.
SO, YOU KNOW, FINALLY
PARTICIPATING IN RESEARCH CAN BE
ABSOLUTELY MEANINGFUL AND
CONTRIBUTE TO SCIENTIFIC
KNOWLEDGE. AND YOU HELP OTHERS.
IT'S ALTRUISTIC. IT'S
PHENOMENAL. BUT IT'S ESSENTIAL
TO APPROACH THE DECISION REALLY
THOUGHTFULLY AND WITH CARE.
>> GREAT. WELL, THANK YOU, ALL,
I SO APPRECIATE YOUR
PARTICIPATING TODAY AND SHARING
YOUR STORIES. I -- SO, THANK
YOU VERY, VERY MUCH. I AM GOING
TO NOW --
>> AUDIENCE: (APPLAUSE).
>> YOU'RE VERY WELCOME.
>> I'M GOING TO PUT TONY ON THE
SPOT AND SEE IF SHE HAS ANYTHING
SHE WOULD LIKE TO ADD AS I
MENTIONED EARLIER, TONY HAS SORT
OF THE POPULATION BASED
KNOWLEDGE AND PERSPECTIVE ON
WHAT IT'S LIKE TO BE A
PARTICIPANT AND THEN FOLKS IF
YOU HAVE QUESTIONS FEEL FREE TO
COME TO THE MICS.
>> GOOD MORNING, I APOLOGIZE FOR
BEING A LITTLE LATE. BUT I JUST
HAVE TO SAY FIRST OF ALL HATS
OFF TO THE FOUR OF YOU. YOU
KNOW, I'M OVERWHELMED. I'M NOT
SURPRISED. YOU GUYS ARE
PHENOMENAL. THANK YOU FOR
PARTICIPATING AND I TOOK A
COUPLE NOTES. SOME OF THESE
THINGS YOU ALL HAVE SAID
BEAUTIFULLY AND I CAN'T SAY THEM
ANY BETTER I DID THE ROLE OF THE
PATIENT REPRESENTATIVE HERE FOR
NINE YEARS AND SO MUCH I LEARNED
THAT I LIKE TO SHARE TO FOLKS
THAT I THINK IT'S HELPFUL TO
KEEP IN MIND AND ONE IS WE ASK A
LOT OF THEM. IT'S NOT EASY TO
BE ON A CLINICAL TRIAL. AND
KEEP THAT IN MIND AS THEY HAVE
MENTIONED SO NICELY WE INVITE
THEM IN. WE HAVE SOMETHING WE
WANT TO OFFER BUT WE'RE ASKING
THEM TO CHANGE THEIR LIVES TO
CONTRIBUTE TO SCIENCE. SO
REMEMBER THAT. THAT WE ARE
ASKING A LOT OF THEM. SOME OF
THE THINGS WE ASK OF THEM
THEY'RE FINE WITH BUT SOME
THINGS ARE CHALLENGING AND SO IF
WE CAN KEEP THAT IN THE BACKS OF
OUR MINDS OR IN THE FOREFRONT
ACTUALLY AT ALL TIMES THAT WOULD
BE -- HELP THE RELATIONSHIP.
THE POWER OF COMMUNICATION AND
THEY'VE TALKED ABOUT THIS SO
MUCH. THIS IDEA OF EXPLAINING
THINGS THAT YOU'RE DOING THAT
COME SECOND NATURE TO US AS THE
PROVIDERS AND THE CLINICIANS.
BUT NOT TO THEM. EVEN THE
SIMPLE EXAMPLE AND THE BEAUTIFUL
EXAMPLE THAT SHOW MA GAVE THAT
ONCE SHE COULDN'T SEE TO TELL
SOMEONE WHAT YOU'RE DOING. IT
SEEMS SMALL BUT HAS SUCH A HUGE
IMPACT AND CREATING AN
ENVIRONMENT OF COMFORT FOR
PATIENT TO GIVE YOU FEEDBACK.
THERE IS STILL A POWER
DIFFERENTIAL WHEN YOU ARE THE
TEAM AND THEY ARE THE PATIENT.
PARTICULARLY BECAUSE PATIENTS,
THEY WANT WHAT YOU HAVE TO OFFER
SO THEY DON'T ALWAYS WANT TO
COME FORWARD AND GIVE YOU SOME
FEEDBACK THAT MAY INDICATE THAT
YOU'RE NOT AS FANTASTIC AS YOU
THINK YOU ARE. BUT IF WE CAN
CREATE THE ENVIRONMENT THAT
SAYS, IN ORDER FOR US TO GET
BETTER WE HAVE TO HEAR FROM YOU
BECAUSE YOU HAVE EYES DIFFERENT
THAN OURS SO I WOULD ENCOURAGE
YOU TO CREATE THAT ENVIRONMENT
THAT WE'RE NOT GOING TO GET
ANGRY OR RETALIATE IF YOU TELL
US WE MESSED UP WE WANT TO KNOW.
WE WANT INPUT ON HOW TO MAKE THE
EXPERIENCE BETTER FOR YOU AND
FOR FUTURE PARTICIPANTS. SO,
AGAIN, THAT PIECE OF
COMMUNICATION. LISTENING TO THE
PATIENT. YOU SAID THAT SO WELL
THEY ARE THE EXPERTS. I'VE BEEN
A NURSE FOR ONE HUNDRED YEARS.
I'VE BEEN AT THE CLINICAL CENTER
FOR 37 AND I CAN REMEMBER AT THE
BEDSIDE COMING HERE, EVERYONE
DOESN'T HAVE A RARE DISEASE BUT
MANY PEOPLE DO AND THEY KNOW IT
BETTER THAN YOU KNOW IT. TRUST
WHAT THEY KNOW. LISTEN TO YOUR
PATIENT. THEY CAN GIVE YOU SO
MUCH INFORMATION TO MAKE YOU
BETTER PROVIDE IRS ERS.
REMEMBER THEY HAVE A LIFE. LIAM
YOU SAID. AS MUCH AS YOU CAN BE
FLEXIBLE WITH ME.
THE BETTER THAT RELATIONSHIP,
NOW THERE ARE SOME THINGS THAT
YOU HAVE DEADLINES AND HARD
STOPS. THEY GET THAT. BUT
THERE ARE TIMES WHEN YOU CAN BE
FLEXIBLE AND SOMETIMES WE
FORGET, AND WE DON'T ENGAGE THAT
CREATIVE PART OF THE PROCESS.
THIS IDEA OF PARTNERSHIP. SO WE
SAY WE'RE PARTNERS, WE HAVE TO
BEHAVE LIKE WE'RE PARTNERS THAT
MEANS RESEARCH TEAMS AND THE
CLINICAL CENTER BECAUSE THE
CLINICAL CENTER'S THE
INFRASTRUCTURE. WE'RE THE ONES
HELPING YOU TO GET YOUR STUDY
DONE SO TALKING AMONGST EACH
OTHER ABOUT HOW YOU CAN MAKE THE
EXPERIENCE BETTER FOR PATIENTS I
THINK WE CAN ALWAYS IMPROVE
AROUND THAT. NEEMA MENTIONED
THIS IDEA OF FEELING TRAPPED IN
THERE BECAUSE THERE'S SOMETHING
THAT CAN BE NEGOTIATED AND
FIGURED OUT. I DON'T KNOW BUT I
AM SAYING THAT OFTENTIMES
BECAUSE WE HAVE 18 INSTITUTES
RUNNING STUDIES HERE WE BECOME
SILOED AND WE DON'T ALWAYS
ENGAGE AROUND WHAT WE MIGHT DO
TOGETHER. I WOULD SAY AS STUDY
TEAMS BE TALKING TO THE CLINICAL
CENTER OR LEADERSHIP ABOUT WHAT
WE MIGHT DO OR IF YOU IDENTIFY,
HM, I WONDER IF WE CAN GET THIS
THING, WE MIGHT BE ABLE TO GET
IT BUT MAYBE THE CLINICAL CENTER
HASN'T HEARD ABOUT THAT. BE
WILLING TO SHARE THAT
INFORMATION AND THEN THE LAST
THING I WOULD JUST SAY IS,
ALWAYS THANK THEM FOR THEIR
PARTICIPATION. WHENEVER
PATIENTS WILL COME TO ME WITH A
CONCERN OR EVEN A COMPLAINT, I
TRY TO SAY TO THEM, THANK YOU
FOR BEING COURAGEOUS ENOUGH TO
GIVE US THE FEEDBACK. AGAIN,
THAT'S THE IDEA OF CREATING THE
ENVIRONMENT THAT IT'S OKAY TO
TELL US HOW TO GET BETTER SO
SAYING TO THEM, THANK YOU FOR
TAKING THE TIME BECAUSE WE KNOW
YOU'RE NAVIGATING A LOT AND SO
TO DO THAT, REALLY TAKES AN
EXTRA STEP SO, THANK YOU ALL,
AGAIN, FOR YOUR PARTICIPATION
AND JUST FOR BEING A PART OF
THIS NIH FAMILY AND THAT'S IT.
>> THANK YOU. QUESTIONS HERE,
UNFORTUNATELY MY COMPUTER DIED
SO I DON'T HAVE ANY QUESTION --
I HAVE ONE QUESTION FROM THE
VIDEO THAT I WILL ASK. AND ALEX
AND GOING TO MONITOR FOR ME. GO
AHEAD, JONATHAN.
>> AUDIENCE: HI. OH, THAT WAS
LOUD. THANKS FOR COMING OUT.
THAT WAS REALLY, REALLY
INFORMATIVE. I WAS WONDERING IF
YOU COULD SAY A LITTLE BIT
ABOUT, SO THIS IS FOR ANYONE FOR
WHOM IT'S RELEVANT IF YOU COULD
SAY A LITTLE BIT ABOUT THE
EXPERIENCE OF SEEING A DOCTOR
AND SORT OF THEM NOT KNOWING
WHAT IS GOING ON. BECAUSE THAT
SEEMS LIKE IT WOULD BE, WELL, I
IMAGINE IT WOULD BE SCARY. BUT
I'M ALSO JUST CURIOUS, YOU KNOW,
HOW YOU NAVIGATED THAT. AND I'M
ALSO CURIOUS IF YOU HAVE
RECOMMENDATIONS FOR DOCTORS
ABOUT HOW THEY SHOULD APPROACH
THOSE KINDS OF ISSUES. YOU
KNOW, LIKE HOW TACTFUL THEY
SHOULD BE OR IF THEY SHOULD JUST
SORT OF SAY I HAVE NO IDEA
WHAT'S GOING ON. YEAH.
HOPEFULLY THOSE QUESTIONS MAKE
SENSE. THANKS.
>> ANYONE WANT TO TAKE THAT ON?
>> YEAH, I WILL.
>> GO AHEAD, ELLEN.
>> WHEN THEY FIRST EXPLAINED THE
PROTOCOL TO ME IN-PERSON MY
DOCTOR WAS VERY HONEST IN
SAYING, YOU KNOW, WE'RE NOT
SURE. THIS IS WHAT A CLINICAL
TRIAL IS ABOUT. WE'RE NOT SURE
WHAT'S GOING TO HAPPEN. BUT WE
APPRECIATE YOU COMING HERE TO
HELP US FIND OUT WILL THIS WORK.
SO HE WAS RIGHT UP THERE RIGHT
AWAY WITH NO, I DON'T KNOW
EVERYTHING. YOU KNOW? YOU
NEVER FEEL GREAT WHEN YOU GO
THROUGH A DOCTOR AND THEY SAY,
WELL, WE'VE NEVER SEEN THIS
BEFORE. THESE ARE NOT WORDS
THAT ARE ENCOURAGING BUT BEING
HONEST ABOUT, WELL, YOU KNOW, I
DON'T KNOW WHAT TO DO YET BUT
WE'RE GOING TO TRY AND FIND OUT
TOGETHER. THAT WAS AN IMPORTANT
PART FOR ME AND, YOU KNOW, I
HAVE DEVELOPED A FRIENDSHIP WITH
MY DOCTOR AND I SAW HIM OUTSIDE
THE CLINICAL CENTER AND HAD
LUNCH WITH HIM. HE BECAME A
FRIEND AFTER THE MANY TIMES I
CAME HERE TO SEE HIM HE CAME TO
SEE ME ONCE IN MY WORLD SO THAT
MAKES A DIFFERENCE TOO.
>> ANYONE ELSE WANT TO TAKE THAT
ON?
>> SURE I WILL TAKE IT ON TOO.
THANKS FOR THAT QUESTION. I
THINK FROM MY CASE WE DON'T
NECESSARILY HAVE SOMETHING TO
HELP ME OUT AND I THINK THE LAST
TWO OR THREE YEARS I GOT REALLY
FRUSTRATED BECAUSE IT BECAME
SCRIPTED HEY, WE HAVEN'T FOUND
ANYTHING YET AND THE THING THAT
MADE IT DIFFERENT FROM MY
PHYSICIAN I HAD THE SPACE TO SAY
I WANT TO PREVENT IT FROM
GETTING WORSE. I FEEL IT. IT'S
GOING DOWNHILL AND FOR HIM
GIVING ME THE CHANCE TO
COMMUNICATE WAS HUGE. IT WAS
EXTREMELY SCARY FOR ME I NEVER
WANT TO HURT PEOPLE'S FEELINGS
BUT IT WAS HARD HEARING THE SAME
THING OVER AND OVER AGAIN EVERY
YEAR. THANKS FOR COMING BUT
THERE STILL HASN'T BEEN ANYTHING
TO HELP YOU OUT YET. BUT
REMINDING ME IN SOMETHING THAT
HE DOES ALL THE TIME AND I THINK
THAT HELPS ME AGAIN SHINE MY
HOPE LIGHT IS SAYING, HEY, HAVE
YOU HEARD, FOR THIS PARTICULAR
EYE PATIENT WE'VE BEEN ABLE TO
FIND A STEM CELL RESEARCH THAT
IS HELPING THEM AND ONE DAY THIS
CAN HELP YOU KIND OF MAKING IT
LIKE THERE IS OTHER OPTIONS,
THERE'S SOMETHING OUT THERE,
AGAIN, BEING OPEN AND HONEST AND
ALLOWING ME TO VENT AND DUMP ON
HIM AS WELL WAS I THINK A HUGE
THING AND IT MADE ME LIKE, OKAY;
THIS IS A PROCESS. IT MIGHT NOT
HAPPEN OVERNIGHT. IT MIGHT BE
FIVE YEARS OR TEN YEARS FROM NOW
BUT HE'S BEING HONEST WITH ME IS
A HUGE, HUGE THING AND THAT'S
HARD SOMETIMES OF NOT HAVING AN
ANSWER. I'M VERY FRUSTRATED
BECAUSE IT TAKES A LOT OUT OF
OUR DAILY LIVES AND WE DO WANT
TO BE LIKE THAT, OH, WE FOUND
THAT SPECIAL, SPECIAL TOOL TO
HELP ME BUT I THINK THAT'S
HONEST THAT YOU'RE ASKING THAT
BECAUSE A LOT OF TIMES IT'S
SCARY TO SAY HEY, I'M REALLY
FRUSTRATED BUT, YEAH, THAT'S
KIND OF MY SENSE OF BEING OPEN,
HONEST AND SAYING, YOU KNOW WHAT
YOUR CASE MAY NOT BUT THERE'S
SOMETHING SIMILAR TO IT THAT IS
HOPEFUL AND THERE'S EXCITEMENT
FOR THAT PARTICULAR SCIENTIFIC
DISCOVERY THAT THEY MADE. THAT
HELPS.
>> I THINK I CAN ALSO ADD A
LITTLE BIT. WHEN YOU HAVE A
RARE DISEASE YOU COME ACROSS
LOTS OF DOCTORS WHO DON'T KNOW
AND THAT'S OKAY. AND SO I
APPRECIATE YOUR QUESTION. I
THINK AS MY COLLEAGUES ON THE
PANEL HAVE SAID, IT'S OKAY TO
ACKNOWLEDGE IF YOU DON'T KNOW SO
BE HONEST, RIGHT? AND THE THING
THAT'S FUNDAMENTAL. I'VE HAD
DOCTORS WHO TOLD ME OH, YEAH, I
HAVE SEEN LOTS OF JANSEN'S
PATIENTS AND I KNEW HE WAS LYING
SO I IMMEDIATELY LEAVE THE ROOM.
SO DON'T UNDERESTIMATE THE
PATIENT KNOWS. AND THAT'S
IMPORTANT. AND THAT THE PATIENT
CAN TELL THAT YOU'RE LYING
BECAUSE WE CAN. WE -- THIS
ISN'T OUR FIRST RODEO, YOU'RE
PROBABLY NOT THE FIRST DOCTOR
THAT'S LOOKED AT SOMEBODY'S
IMAGES AND SAID, YOU KNOW,
SOMETHING CRAZY. SO BE HONEST.
YOU KNOW, DOCTORS SHOULD REALLY
OPENLY ACKNOWLEDGE THAT YOU
DON'T HAVE ALL THE ANSWERS AND
THAT'S FINE BECAUSE AS WE JUST
HEARD D HONESTY BUILDS TRUST.
AND LISTEN ACTIVELY. YOU HAVE
TO LISTEN TO THE PATIENT'S
CONCERNS BECAUSE WHEN YOU DO
THAT, YOU ARE GETTING INTO THEIR
SPACE, RIGHT? YOU ARE ENTERING
INTO THAT RELATIONSHIP WITH
THEM.
IT SHOWS THAT YOU VALUE THE
PATIENT'S EXPERIENCE AND ARE
COMMITTED TO FINDING A SOLUTION
AND I THINK THAT COMMITMENT
FINDING ANSWERS IS WHY WE KEEP
COMING BACK TO A DOCTOR. RIGHT?
THE DOCTOR SHOULD EXPRESS THAT
WILLINGNESS TO INVESTIGATE
FURTHER. OR TO -- OR SAY HEY,
I'M GOING TO CONSULT WITH
SOMEBODY, LOOK AT THE MEDICAL
LITERATURE, REFER YOU TO
SPECIALISTS IN THE FIELD.
THAT'S IMPORTANT BECAUSE THEN IT
SHOWS YOU ENTERED INTO OUR WORLD
IF YOU SAY I DON'T KNOW AND STEP
BACK IT SHOWS THAT YOU DON'T
CARE. WE CAN PICK UP ON THAT
INSTANTLY AND THE DOCTORS THAT
ENTER INTO RELATIONSHIPS WITH
PARENTS AND ENGAGE IN
PARTNERSHIPS, THEY ENGAGE IN
COLLABORATIVE LEARNING
EXPERIENCES. SO WE ARE THEN A
TEAM. WE BEGIN TO WORK TOGETHER
TO ACCESS DATABASES, REGISTRIES,
PATIENT ADVOCACY ORGANIZATIONS
AND WE FIND INFORMATION THAT
BECOMES VALUABLE AND SUPPORTIVE
AND THAT WHOLE IDEA OF
MAINTAINING OPEN COMMUNICATION
FOLLOW UP, YOU KNOW, WHAT DID --
FOLLOW UP AND SAY IT, WHAT DID
YOU LEARN? WHAT DID YOU FIND
AND HOW CAN WE THEN ENCOURAGE
PATIENTS' ENROLLMENT AND EMPOWER
PATIENTS IN THEIR OWN JOURNEY
THIS IS A COLLABORATIVE PROCESS
AND THE UNKNOWN IS IMPORTANT
BECAUSE WE ARE ON A JOURNEY OF
DISCOVERY TOGETHER AND I THINK
IN THAT JOURNEY WE ALSO HAVE TO
DOCUMENT FINDINGS. IT'S REALLY
IMPORTANT TO SAY, YOU KNOW, KEEP
DETAILED NOTES OF WHAT WE FOUND
THAT DIDN'T WORK. WHAT NEW
FINDINGS CAME ACROSS THAT WE
BOTH HAVE BEEN ON TOGETHER AND I
THINK THAT IS WHAT CREATES A
LONG-TERM RELATIONSHIP AND
PARTNERSHIP THAT WILL EVENTUALLY
LEAD TO SOLVING SOME OF THESE
MYSTERIES.
>> GREAT. THE ONE QUESTION THAT
WE HAD FROM THE DISCUSSION BOARD
WAS FOR, I'M GOING TO DIRECT TO
YOU, NINA BECAUSE YOU MENTIONED
IT THAT WHEN YOU AND YOUR SONS
WERE HERE FOR A WHOLE WEEK THERE
WERE LIMITATIONS ON YOUR ABILITY
TO LEAVE THE CAMPUS. WHAT'S THE
ANSWER TO THAT? WHY DID THEY
SAY YOU HAVE TO STAY HERE?
>> I THINK THAT IT'S -- IT WAS
SAFETY. RIGHT? I MEAN, I'M
PRETTY SURE IT'S SAFETY AND IT
WAS SOME OF THESE PREPANDEMIC OR
THE PANDEMIC RULES THAT JUST
DIDN'T HAVE -- THAT DIDN'T
CHANGE AND I THINK MISS TONY
COULD PROBABLY WEIGH IN HERE. I
THINK THERE'S A DISCONNECT HERE
BETWEEN THE TWO TEAMS. THEY
HAVE TO TAKE MORE. WE HAVE TO
COME UP WITH CREATIVE SOLUTIONS.
RIGHT? MY KIDS WERE NOT
STRAPPED TO ANYTHING. THEY WERE
NOT PLUGGED IN OR TIED IN. THEY
COULD LEAVE. RIGHT? AND WE
COULD COME BACK IN THE EVENING
THEY COULD HAVE GONE OUT. WE
COULD HAVE HAD DINNER OUTSIDE.
JUST GETTING OUTSIDE OF THE
CENTER WOULD BE VERY WELCOMING
AND I KNOW SOMEBODY MENTIONED ON
THE FLOOR THAT HEY, WE USED TO
DO THIS. BEFORE THE PANDEMIC
AND THEN THEY SHUT IT DOWN BUT
THEY NEVER REINSTATED. SO THERE
WAS A DISCONTRADICT. WE HAD
SOME INFORMATION FROM ONE OF THE
NURSES THAT YES, IT WAS A
POSSIBILITY.
THEY DIDN'T KNOW WHAT WAS THE
POSSIBILITY AND WE COULDN'T FIND
SINGLE THAT COULD VETO AND IT I
WENT TO THE HEAD HONCHO AND HE
SAID NO WAY ARE YOU LEAVING THE
CENTER. I'M ALSO ON THE
ADVISORY BOARD HERE AND THIS IS
IMPORTANT.
I THINK THAT KIDS NEED TO BE
ABLE TO LEAVE. THAT THEY FEEL
TRAPPED THERE AND WE HAVE TO
FIND A WAY TO DO THAT. WHETHER
IT'S A DAY PASS THEY SAID
WHETHER WE WERE AT THE HOUSE WE
COULD DO THAT BUT WE WEREN'T, WE
WERE -- BECAUSE I HAD TWO KIDS
AND THEY WERE BOTH IN DIFFERENT
ROOMS, THAT IT WAS IMPOSSIBLE
FOR THEM TO LEAVE AND I'M NOT
SURE WHAT THAT MEANS BY THE WORD
IMPOSSIBLE, IN OUR WORLD, IS
MEANT TO BE MADE INTO POSSIBLE,
RIGHT? BECAUSE THAT'S THE LIVES
WE LIVE, SO THERE'S GOT TO BE
CREATIVE WAYS TO DO THAT. AND
I'M PRETTY SURE WE CAN DO THAT.
IT'S ABOUT HOW TO DO IT SAFELY
AND MAKE SURE THAT NOBODY ELSE
IS HARMED IN WE DO SO BUT IT HAS
TO HAPPEN.
>> TONY'S GOING TO GIVE US
FEEDBACK ON THAT QUESTION.
>> I THINK THIS IS ONE OF THOSE
EXAMPLE WHERE IS THE RULES AND
REGULATIONS CAN BUMP UP AGAINST
THE DESIRES.
AND AS POINTED OUT ABSOLUTELY
THE PANDEMIC RESTRICTIONS WERE A
HUGE PART OF THE INABILITY TO
ALLOW A PAS WHICH IS SOMETHING
THAT USED TO HAPPEN IN THE
CLINIC AND THAT THEY DON'T WANT
TO REENGAGE. SO THAT'S ONE
PIECE WHERE YOU'VE GOT A RULE
AND YOU'VE GOT TO TRY TO FIGURE
OUT HOW TO NAVIGATE.
AND SOMETIMES IT'S NOT THE
ANSWER WE WANT. THAT DOES
HAPPEN. ALTHOUGH I DO THINK
THAT IDEA OF HAVING
CONVERSATIONS BETWEEN TEAMS AND
CLINICAL CENTER SO YOU CAN START
THINKING ABOUT AND PLANNING
AHEAD. HOW DO YOU -- MAYBE YOU
CAN DO SOMETHING NUANCED FOR A
CHILD OR A CERTAIN POPULATION.
WHERE YOU'RE NOT BREAKING THE
ENTIRE RULE OF MAINTAINING THE
SAFETY BUT WHEN YOU COME UP ON
IT IN THE MOMENT AND YOU DON'T
HAVE A PLAN THERE'S VERY LITTLE
YOU CAN DO BECAUSE THEN EVERYONE
SORT OF BRISTLES AND GET XOOEXCD
AND NERVOUS ABOUT IT AND WE
THINK PROACTIVELY HOW DO WE
THINK ABOUT THIS NEXT YEAR OR
THE NEXT PATIENT THEN WE MIGHT
COME UP WITH SOME CREATIVE
SOLUTIONS.
>> THE PATIENT, SO I THINK I
SAID THIS EARLIER, OUR GUESTS
TODAY ARE FROM THE PATIENT
ADVISORY GROUP FROM THE CENTER
HERE. I HAVE NOW DOUBT THEY'RE
GOING TO PUT THIS ON THE AGENDA
TO EXPLORE FURTHER. SO I JUST
WANT TO THANK ALL OF YOU, AGAIN,
SO MUCH, FOR THE TIME AND EFFORT
AND INCONVENIENCE OF NAVIGATING
THROUGH THE WELCOME CENTER HERE
ON CAMPUS. AND REALLY
APPRECIATE YOUR PARTICIPATION
AND SHARING ALL OF YOUR THOUGHTS
AND COMMENTS.
>> THANK YOU.
>> THANK YOU FOR ALL YOU DON'T
WE WOULDN'T BE HERE WITHOUT ANY
OF YOU.
>> THANKS, EVERYBODY, FOR
JOINING US TODAY. NEXT WEEK IS
INTERNATIONAL DAY SO WE'RE GOING
TO HAVE ROBERT STEELE WHO IS
FROM OUR DEPARTMENT AND THEN TWO
GUESTS, JOE M WHO USED TO BE IN
OUR DEPARTMENT BUT HAS FLED TO
SCOTLAND AND THEN DIR KUS WHO IS
JOINING US FROM KENYA. NEXT
WEEK, SAME TIME, SAME PLACE AND
THAT'S OUR LAST SESSION.
I'M VERY SAD ABOUT THAT. HOPE
TO SEE YOU ALL THERE.