WELCOME EVERYBODY TO THE BRAIN
NEWG WITH THE CONTINUING TRIAL
RESPONSIBILITIES.
FROM THE DIRECTOR OF THE NIH
BRAIN INITIATIVE I WANT TO
THANK EVERYBODY WHO MADE THIS
WORKSHOP HAPPEN.
STARTING WITH CHRISTINE AND
THE COCHAIRS NEWG AND THE REST
OF THE NEWG MEMBERS AND THE
STAFF AT NIH WHO ARE FOCUSED
AND DEDICATED TO ADDRESSING
THESE ISSUES OF NEURAL ETHICS
AND WITH ALL THE WORK TO PUT
THIS TOGETHER.
WE ARE EXCITED TO BRING
TOGETHER COMMUNITIES.
BUT WE ARE MOTIVATED BY THAT
COLLECTIVE SUGGESTION FROM
PEOPLE FROM DIFFERENT DOMAINS.
VERY IMPORTANTLY ARE THE HUMAN
PARTICIPANTS WE REALLY DO SEE
THE NEED TO LOOK AT A SHARED
RESPONSIBILITY TO FACILITATE
RESEARCH RELATED CARE AFTER
THE TRIAL FORMALLY ENDS BUT
QUITE HONESTLY IT'S NOT CLEAR
HOW TO MAKE THIS HAPPEN HOW WE
SPECIFY OR OPERATIONALIZE WHAT
NEEDS TO BE DONE IN REALITY.
SO THE WORKSHOP IS STRUCTURED
THROUGH A SERIES OF FOUR
PANELS.
TODAY WE WILL START WITH THE
FIRST TWO.
WE WILL START WITH THE PANEL
RIGHT AFTER THIS TO EXPLORE
THE NEEDS THE STUDY
PARTICIPANTS HAVE IN RELATION.
THE SECOND PANEL OF THE
AFTERNOON ABOUT THE
STAKEHOLDERS CURRENTLY PROVIDE
AND WHAT IS IN THE REALM OF
POSSIBILITIES FOR WHAT THEY
COULD FACILITATE FOR RESEARCH
CARE.
TOMORROW WE CONTINUE THE
CONVERSATION AND THE THIRD
PANEL SEEK PERSPECTIVES AT A
REASONABLE LEVEL THAT IS
ASSOCIATED AND THEN THE LAST
SESSION WILL FOCUS ON THE
UNMET NEEDS AND HOW IT CAN
FILL THOSE AND HOW I'M LOOKING
FORWARD TO EVERYBODY'S
PARTICIPATION LEVEL TODAY AND
TOMORROW IT WILL HAVE THE
REASONABLE EXPECTATIONS THAT
COULD INFORM GUIDELINES AND
BEST PRACTICES.
THANK YOU FOR BEING HERE THESE
ARE TOUGH QUESTIONS WE ARE
ASKING NOT THE LEAST OF WHICH
OF HOW TO MAKE THINGS HAPPEN
AFTER WE DECIDE WHAT MIGHT
NEED TO HAPPEN AND THEN WILL
DISCUSS THE INTRODUCTION OF
BACKGROUND.
>> THANK YOU.
I WILL START WITH QUICK
INTRODUCTION AND BACKGROUND SO
WE'RE ON THE SAME PAGE FOR THE
REST OF THE WORKSHOP.
FIRST LET ME SPEAK TO WHY WE
NEED THE SCIENCE THAT IS
RELEVANT TODAY DISORDERS CAUSE
A HIGH BURDEN OF DISEASE IN
THE US AND WORLDWIDE THAT
EXCITING PERIOD OF
NEUROSCIENCE THAT SCIENTIST
HAVE DEVICES TO TRACK THESE
CONDITIONS.
HOWEVER THE DEVELOPMENT OF
THEM TO BE USED ON HUMAN
SUBJECTS.
WE CANNOT ADVANCE THE FIELD
WITHOUT THE GENEROUS
CONTRIBUTION THAT PATIENTS
MAKE ON TIFFANY'S TRIALS.
IT IS WORTH NOTING THESE ARE
PATIENTS THEY FOUND THEIR
TREATMENT RESISTANT MAY NOT
HAVE OTHER OPTIONS THEY MAY OR
MAY NOT HELP THESE PATIENTS
THEY CONTRIBUTE TO THESE
TRIALS SO IT IS IMPERATIVE WE
HAVE HIGH ETHICAL STANDARDS.
THIS WORKSHOP IS RELATED TO
THAT GENERAL GOAL OF NIH.
SO SPECIFICALLY WE WILL GET
THE NEEDS THAT PATIENTS MAY
HAVE AFTER THE TRIAL HAS ENDED
WE ARE LOOKING AT RESEARCH
RELATED NEEDS TO THE DEVICE
THAT WAS IMPLANTED AFTER THE
TRIAL HAS ENDED FOR EXAMPLE
PATIENTS MAY USE EXPERIENCE
BENEFITS FROM THE DEVICE AND
THEN THEY WANT TO KEEP IT
AFTER THE TRIAL ENDS.
AND THEN THEY NEED MAINTENANCE
LIKE REPLACING BATTERIES OR
THE EXPECTATION.
SO WITH OTHER DEVICE TRIALS IN
THE NEEDS THAT PATIENTS HAVE
RELATED TO THE TRIALS AFTER
THE TRIAL HAS ENDED.
SO THE CLASSIC SCENARIO IS
ONCE THE TRIAL HAS ENDED BUT
BEFORE IT HAS BEEN APPROVED
FOR CLINICAL USE OF THOSE
SCENARIOS ARE POSSIBLE IF THE
TRIAL DISCONTINUES PREMATURELY
BY THERE IS NO REGULATORY ROLE
FOR THE DEVICE THOSE FORMER
PARTICIPANTS MAY NOT HAVE
COVERAGE OPTIONS WITH THEIR
INSURANCE OR IF AT ANY POINT
IN TIME MAY DISCONTINUE THE
PRODUCT SO I WENT TO HIGHLIGHT
THE CLASSIC SCENARIO IS
TIME-LIMITED THAT AT LAST
UNTIL IT IS APPROVED.
IT MAY LAST LONGER SO SEVERAL
REASONS HAVE BEEN EXPRESSED BY
OTHERS INVOLVED IN THESE
TRIALS MAY HAVE
RESPONSIBILITIES OF ATTENDING
TO THESE NEEDS SO IF YOU ARE
PROFESSIONAL STAKEHOLDER TO
RECEIVE AN EXTRA RENTAL DEVICE
YOU RESPONSIBILITY FOR HELPING
PARENTS TO HARM OF THOSE
INDIVIDUALS WE ALSO HAVE
RESPONSIBILITY TO HELP ADVANCE
PATIENTS INTEREST AND WHILE
OVER TIME THEY DON'T
NECESSARILY STOP AT THE TIME
THE TRIAL ENDS.
IT'S OFTEN REGARDED AND SECOND
AND WITH THAT RECIPROCITY AND
WITH THOSE RISK THEY UNDERTAKE
AS PARTICIPANTS SO FOR EXAMPLE
OF THIS RESEARCH PARTICIPANT
EXPRESSED IT FELT LIKE HE LUCK
IF I IMPLANT THINGS IN MY
BRAIN AND I'M WALKING AROUND
WITH THIS YOU HAVE A
RESPONSIBILITY TO LOOK AFTER
ME.
AND THIRD BUT ALTHOUGH MANY AS
A MEANS TO AN END AND THEN
WITH THE CONCERNS OF THE X PER
AND WITH THAT RELATIONSHIP
BETWEEN THE PARTICIPANTS AND
THOSE INVOLVED IN THE TRIAL
HAS SOME TYPES OF RELATIONAL
DUTIES.
WITH THAT POSTTRIAL
RESPONSIBILITIES HAVE LIMITS
AND OPPORTUNITY COSTS
STAKEHOLDERS ALSO HAVE
IMPORTANT TRIALS WITH THE
POPULATION AT LARGE AND IN THE
MOST EXTREME FORMS THEY MAY
NOT BE ABLE TO CONNECT ANY
TRIALS AT ALL FOR THEIR CARE
NEEDS.
SOME PREFER TO ORDER MORE THAT
FUNDING THAT IS MEANT FOR
RESEARCH SO I WILL SAY THIS
ARGUMENT IS IN THE CONTEXT OF
THE IMPLANTED DEVICES IT'S
OBVIOUS BETWEEN RESEARCH AND
CLINICAL CARE.
SOME WOULD ARGUE SO CONSENTING
TO A LACK OF INFORMATION.
AND THIS LACK OF ARRANGEMENT
IS ACCEPTABLE.
AND THEN HOLDING A WORKSHOP IN
2017 WITH TOPICS AROUND CARE.
BUT THEN TO GO THROUGH THE
FRAMEWORKS AND STANDARD
PRACTICES.
AND THEN WITH THE MEDICAL
DEVICES AND THE NEWG WORKING
GROUP DRAFTED THE FOLLOWING
TREATY OF THINGS TO CONSIDER.
FIRST PROCESSED TRIAL NEEDS
SHED ANTICIPATE AHEAD OF THE
TRIAL IN DIFFERENT SCENARIO
SHOULD BE PLANNED FOR.
SECOND.
PROFESSIONAL STAKEHOLDERS HAVE
A LIMITED SHARED
RESPONSIBILITY TO TAKE
REASONABLE STEPS TO FACILITATE
CONTINUED ACCESS TO DEVICES
THAT BENEFIT PARTICIPANTS AND
THEY MAY HAVE FURTHER
RESPONSIBILITIES.
FINALLY PATIENTS SHOULD BE
INFORMED ABOUT POSTTRIAL NEEDS
AND PLANS.
I WENT TO EMPHASIZE TWO
THINGS.
FIRST IN THIS WORKSHOP THE
RESULTS OF WHICH WERE
PUBLISHED IN THERE APPEARS TO
BE A CONSENSUS IN THE FIELD OF
THESE THREE POINTS SO WE WOULD
LIKE TO TAKE THESE THREE
POINTS AND BASICALLY START
FROM THERE.
THIS ALSO MEANS WE WOULD LIKE
TO LIMIT THE TIME SPENT WITH
THE APPLICABLE FOUNDATIONS OF
THE STATEMENTS AND THE THING
IT MADE PROGRESS ON THE
SPECIFICATIONS.
SO IT'S CURRENTLY GOING ON IN
THE FIELD THERE IS STILL SOME
VARIETY AND THE EXTENT OF
POSTTRIAL CARE AND WHAT THIS
ENTAILS.
BUT THAT IS NOT SURPRISING
CONSIDERING FROM THE WORKING
GROUP I JUST QUOTED WAS PRETTY
HIGH LEVEL IT DOESN'T REALLY
SAY THAT THIS IS APPROPRIATE
BUT TO ENCOURAGE TO MAKE PLANS
AND THAT HELPS WITH THEIR
LONG-TERM CARE AND SOME FORMER
PARTICIPANTS TO EXPERIENCE
ANXIETY AROUND THE UNCLEAR
INDIAN ACCESS TO THE DEVICE
FOLLOW-UP CARE AND MAINTENANCE.
EVEN A LOSS OF ACCESS TO
BENEFICIAL DEVICES.
SO THE VERY HELPFUL FOR THE
FIELDS RELATED TO
SPECIFICATION AND
IMPLEMENTATION OF THOSE
HIGHER-LEVEL PRINCIPLES I JUST
OUTLINED WHAT ARE THOSE
STAKEHOLDERS TO PROVIDE.
AND HOW ARE THEY PROVIDED FOR
AND WITH THOSE DIALOGUE TO THE
STAKEHOLDER AND IN ORDER TO
PREPARE FOR IT.
AND SOME OF THE MEMBERS IF
THERE WERE POINTS THAT ARE NOT
BEING BROUGHT UP THEN STAFF
MEMBERS WILL MAKE SURE TO ADD
THESE TO THE CONVERSATION.
THE GOAL OF THE WORKSHOP IS TO
TALK REASONABLE EXPECTATIONS
FOR THAT CARE PLANS THAT CAN
INFORM BEST PRACTICES IN THE
COMMUNITY EVENTUALLY.
NOW I JUST WANT TO HIGHLIGHT
THE FOUR PANELS THAT WILL
HOPEFULLY BUILD ON EACH
OTHER.
FIRST WE WILL IDENTIFY THE
POSTTRIAL NEEDS AND IN THOSE
ARE CURRENTLY OVER ALREADY
COVERED IN TOMORROW WE WILL
TAKE THIS LIST OF NEEDS AND
SAY WHAT IS THE MINIMUM OF THE
RESEARCH RELATED CARE?
AND THEN FINALLY THE
DIFFERENCE BETWEEN PANEL
NUMBER THREE INTO WHAT SHOULD
BE FACILITATED AND HOW DOES IT
FILL THE GAPS?
THANK YOU AGAIN FOR BEING HERE
AND SPECIAL THANKS TO THE NIH
PLANNING COMMITTEE WHO HAVE
LED THIS EFFORT.
I WILL HAND IT OVER TO PANEL
NUMBER ONE.
>> THANK YOU SO MUCH.
WE WILL KICKOFF PANEL NUMBER
ONE SO LOOKING AT THE RELATION
TO THEIR TRIAL PARTICIPATION
AFTER THE TRIAL ENDS.
THE FIRST SPEAKER IS
MISS BRANDY ELLIS WHO IS A
PATIENT PARTICIPANT FROM THE
DEVICE CLINICAL TRIALS IN
FIRST ENROLLED IN 2011 AS PART
OF THE PHYSICIAN SPONSORED
EXPERIMENTAL STUDY THAT
INVOLVES DEEP BRAIN
STIMULATION FOR TREATMENT
RESISTANT DEPRESSION USING
DEVICES.
SINCE THEN SHE HAS RECEIVED
ONGOING CARE AND NEGOTIATES
WITHOUT IT FOR HER DEVICE
MAINTENANCE.
>> IT AFTERNOON.
IT IS A PLEASURE TO BE HERE.
I WILL BREEZE THROUGH THE
INTRODUCTION BECAUSE I CAN
TALK ABOUT THIS FOR HOURS SO
WE WILL GO TO THE NEXT SLIDE
AND YOU CAN SEE THIS IS WHAT
IS CURRENTLY IN MY BRAIN.
BEFORE YOU ASK WHY SOMEBODY
WOULD DO THIS, LET'S GO TO THE
NEXT SLIDE, YOU WILL SEE WHY.
I LITERALLY HAD TRIED
EVERYTH EVERYTHING.
IF A DOCTOR COULD THINK OF IT
I WOULD TRY IT.
FOUR YEARS.
WAS COMPLETELY DISABLED.
NOTHING HAS WORKED.
AT THAT POINT I DECIDED IF I
COULD NOT FIND DATA ON WHAT
WORKED FOR TREATMENT RESISTANT
DEPRESSION BECAUSE EVERYTHING
WAS GOAL ORIENTED TO PEOPLE
WHO WERE FEELING LITERALLY
EVERYTHING.
AND I HAD A BELIEVE THAT
NOTHING WOULD WORK BUT STILL
IT WOULD BE BENEFIT DESIGNED
FOR THE PEOPLE WHO COME AFTER
ME SO IF YOU GO TO THE NEXT
SLIDE THIS IS A STUDY I WAS IN
AND THEN GO TO THE NEXT SLIDE
YOU CAN SEE IT IS A REALLY
LONG RECOVERY PROCESS.
I AM VERY THANKFUL IT DID WORK
AND IN THE NEXT SLIDE IT WAS
YEARS FROM THE TIME I DEVICE
WAS IMPLANTED UNTIL I FELT
WELL.
THIS IS NOT A QUICK FIX.
AS PATIENT YOU ARE REALLY
COMMITTING TO THE LONG HAUL
AND FOR ME BECAUSE MY
DEPRESSION WAS SO SEVERE, THE
INITIAL WE NEED YOU TO NOT DIE
FOR THE FIRST YEAR PLEASE YOU
WILL MESS UP OUR EXPERIMENT.
THEY DID NOT SAY THAT BUT
THAT'S HOW SOMEBODY WOULD
INTERNALIZE IT.
IT WAS QUITE A COMMITMENT IF
YOU GO TO THE NEXT SLIDE THESE
ARE THINGS I HAVE EXPERIENCED
OUR FRIENDS HAVE EXPERIENCED
AND THIS LEADS ON WHAT I WOULD
LIKE OF POSTTRIAL CARE.
I WILL GO SPECIFICALLY INTO
SOME OF THE SUGGESTIONS THAT I
HAVE I HAD BATTERY
REPLACEMENTS MY BATTERIES WERE
LASTING BETWEEN 11 AND 13
MONTHS WITH ORIGINAL SETTINGS.
I WAS IN MY THIRTIES.
SO POTENTIALLY FACING 30 OR 40
SURGERIES FOR BATTERY
REPLACEMENTS CUTTING INTO THE
SAME AREA.
NOW I HAVE A RECHARGEABLE
BATTERY BUT IT IS NOT FDA
APPROVED SO THERE IS A RISK I
HAVE TO GO BACK TO A STANDARD
BATTERY AND THOSE ANNUAL
SURGERIES.
I HAVE HAD VARIOUS
MALFUNCTIONS WITH THE
IMPLANTED DEVICE OR THE DEVICE
THAT I USED TO RECHARGE.
AND I AM STILL ENGAGING AND
FOLLOW UP AND I WILL HELP THEM
AS LONG AS THEY FIND MY HELP
BENEFICIAL.
THERE ARE OTHER THINGS I WOULD
LIKE TO SEE IMPROVED GENERAL
SECURITY WERE GETTING TO WHERE
EVERYTHING IS TRACKABLE AND
I'M DEPEND UPON THE FACT THAT
THOSE PEOPLE CLOSE TO ME WANT
TO KEEP ME AND WILL NOT BE
HACKING MY DEVICE.
SO MY FIRST SURGERY WAS
ACTUALLY COVERED BY MY PRIVATE
INSURANCE AND IT IS MY
UNDERSTANDING THE FIRST
PARTICIPANT WHO HAD THEIR
INSURANCE COVER THE INITIAL
PROCEDURE.
I HAVE TO ASSUME THAT WAS IN
PART BECAUSE OF THE LIST OF
ALL OF THE THINGS I HAVE TRIED
WAS VERY EXPENSIVE.
NOTHING WAS WORKING.
SO THIS WAS CONSIDERED A
CHANCE FOR ME TO SURVIVE WITH
LESS EXPENSE AND IT HAS WORKED
MY MEDICAL CARE EXPENSES
PLUMMETED ONCE I WAS NOT
NEEDING AS MUCH CARE.
BUT ONGOING SURGERIES AND
THOSE SORTS OF ISSUES IS I CAN
UNDERSTAND THE INITIAL TRIAL.
THAT IS THE EXPERIMENT I
COMPLETELY UNDERSTAND WHY
INSURANCE WOULD NOT COVER
SOMETHING EXPERIMENTAL.
BUT FOR ME HAVE HAD THIS
DEVICE FOR TEN YEARS IT IS NOT
AN EXPERIMENT FOR ME ANYMORE.
WE KNOW THIS WORKS THIS IS THE
ONLY THING THAT DID WORK AND
IF I NEED TO HAVE BATTERY
REPLACEMENT OR ANYONE OF THOSE
THINGS, IT'S A WAY TO KEEP ME
ALIVE IT IS NOT AN EXPERIMENT
ANYMORE WE KNOW THIS WORKS I
HAD UNFORTUNATE BLINDED
INCIDENT I DID NOT KNOW HIS
OFF FOR A COUPLE OF WEEKS AND
I GOT UNWELL AND THEN WE FOUND
OUT IT WAS OFTEN THAT WE FOUND
OUT IT REALLY DOES WORK AND I
REALLY DO NEED IT.
THE SECOND THING I WOULD FOCUS
ON IS COMPATIBILITY.
I AM CURRENTLY USING A
RECHARGEABLE BATTERY AT THIS
POINT IN TIME HAS SAVED ME SIX
SURGERIES.
BUT THAT BATTERY IS NOT FDA
APPROVED.
MOST PEOPLE WHO GET DBS GET IT
FOR PARKINSON'S AND THEY ARE
OLDER AND THE DEVICE DOES NOT
DRAIN THE BATTERY AS QUICKLY
SO THEY WOULD NOT NEED SURGERY
EVERY SINGLE YEAR.
MAYBE RECHARGEABLE IS NOT AS
NECESSARY FOR THEM BUT FACING
30 OR 40 SURGERIES OVER MY
LIFE EXPECTANCY THAT IS A BIG
DEAL THEY DON'T HAVE A BATTERY
THAT IS FDA APPROVED AND WENT
TO DISCONTINUE PRODUCTION.
SO WHAT I WOULD LIKE IS TO
HAVE INCENTIVES TO MAINTAIN
COMPATIBILITY.
IF YOU GO TO THE NEXT SLI
SLIDE, THERE ARE MULTIPLE
DEVICE MANUFACTURERS BUT THERE
IS NO INCENTIVE FOR ONE
COMPANY TO INVEST IN MAKING
THEIR BATTERY COMPATIBLE WITH
EVERYBODY OR IF THEY WANT TO
START MAKING THIS BATTERY THAT
THERE IS AN APPROPRIATE
SUBSTITUTION.
BECAUSE EXPECTING SOMEONE WHO
IS RELYING ON A DEVICE TO LIVE
AND HAVE AN OPERATION EVERY
YEA YEAR, EVEN IF THIS
BATTERY ENDED AFTER FIVE YEARS
THAT IS STILL A NUMBER OF
SURGERIES ALSO I HAVE HAD
DEVICE FAILURES WITH MY
RECHARGER AND I AM VERY
CONCERNED AT SOME POINT SO I
WOULD LIKE TO QUICKLY BRING UP
THE NEXT SLIDE WHICH IS MY
WISH LIST AND THAT OVERCOMES
THOSE SUGGESTIONS AND THEN WE
CAN GET INTO QUESTIONS.
MY WISH LIST WOULD INCLUDE
THAT I WAS FORTUNATE I HAD
PARENTS THAT WERE WILLING TO
SUPPORT ME WHEN I WAS DISABLED
FROM THE DEPRESSION AND HELPED
I HAD PRIVATE HEALTH INSURANCE
FROM THEM.
I HAD THE ABILITY TO BE
UNEMPLOYED AND HAVE THEM
SUPPORT ME.
IF YOU REQUIRE PARTICIPANTS TO
FOOTBALL THE BILLS THEMSELVES
TO LIVE THEN YOU ONLY GET
PARTICIPANTS LIKE ME WHO HAVE
AN AFFLUENT FAMILY AND CAN
AFFORD TO SUPPORT THEM WHILE
THEY ARE IN THE TRIAL.
AND THAT IS NOT GOOD FOR
ANYBO ANYBODY.
WE NEED MORE DIVERSE PEOPLE
WITH THESE TRIALS AND TO MAKE
THAT HAPPEN THERE HAS TO BE A
WAY TO GIVE THEM SUPPORT.
I WAS DISABLED AND NOT WORKING
BUT CHILDCARE, ANY OF THOSE
MIGHT HAVE MADE THE DIFFERENCE
IN MY RECOVERY.
I WAS VERY FORTUNATE BUT WE
NEED PEOPLE ACROSS ALL
DIFFERENT BACKGROUNDS TO
PARTICIPATE IN THESE TRIALS
AND SUPPORT IS ONE OF THOSE
THINGS THAT IS NECESSARY.
THE NEXT ISSUE OF GUARANTEED
COVERAGES LIKE I SAID I AM
RELYING ON THIS DEVICE.
THE LIKELIHOOD THAT I SURVIVE
IF I CANNOT USE THIS ANYMORE
IS NOT 100 PERCENT SO I AM
CONCERNED I WILL NEVER KNOW
FROM ONE SURGERY TO THE NEXT
IF THE NEXT ONE WILL BE
COVERED BY INSURANCE.
I WILL NEVER KNOW IF I GET
INTO A CAR ACCIDENT IF THE
BRAIN SURGERY IS COVERED BY MY
INSURANCE OR FACE THE PROSPECT
OF NO LONGER HAVING THIS
DEVICE.
I WOULD LIKE THERE TO BE AWAY
TO SAY YOU DID THIS EXPERIMENT
AS PART OF THE CLINICAL TRIAL
THAT WAS APPROVED AND SINCE IT
WORKS FOR YOU THIS IS NO
LONGER AN EXPERIMENT FOR YOU.
I HAVE NO CONTROL.
PERPETUAL POINT OF CONTRACT
THEY HAVE SUPPORTED ME FOR ALL
TEN YEARS WITHOUT QUESTION FOR
30 YEARS FROM NOW WHEN I'M IN
THE RETIREMENT HOME I DON'T
KNOW WHO WILL BE RESPONSIBLE
FOR CHECKING MY DEVICE
SETTINGS.
AND I WOULD VERY MUCH LIKE
THERE TO BE A REQUIREMENT IF
IT'S IN MY HEAD AND IT WORKS
TO SAY WE'VE DECIDED NOT TO
MAKE THIS ANYMORE AND NOT TO
COVER THIS ANYMORE THAT IF YOU
STOP MAKING THIS BATTERY THERE
HAS TO BE A COMPATIBLE
ALTERNATIVE AND UNTIL THERE IS
YOU CANNOT ABANDON THE
PATIENT'S SO SOME MINOR THINGS
I WOULD LIKE AS FAR AS
DOCUMENTATION I THINK A
REGISTRY OR SOMETHING THE
MATTER WHERE YOU ARE SOMEBODY
CAN FIND OUT THE SETTINGS IF I
AM TIRED OF PORTUGAL I WILL
NEED A SHEET OF PAPER TO TAKE
TO A DOCTOR TO SAY THIS IS
WHAT I HAVE IN THESE ARE THE
SETTINGS AND THIS IS WHAT YOU
NEED TO VERIFY.
I THINK PARTICIPANTS SHOULD
AUTOMATICALLY GET ACCESS TO
ALL PUBLICATIONS.
BECAUSE I'M ONE OF THE PEOPLE
THAT READS ALL OF THE
PUBLICATIONS.
THAT IS MY RECOMMENDATIONS
THOSE THAT HAVE DONE FOLLOW UP
AND I'M HAPPY TO ANSWER ANY
QUESTIONS IF ANYBODY HAS ANY.
>> WE HAVE TIME FOR ONE
QUESTION AND THEN WE CAN GET
INTO THE ISSUES BUT THE
QUESTION.
>> 90 PERCENT IS A THANK YOU
FOR CONVEYING THE STARTS WE
HAVE PLENTY OF WORK TO DO AND
AMAZING IT IS TO FOLLOW UP ON.
IT'S VERY COURAGEOUS OF YOU.
THANK YOU.
I'M HAPPY YOU HAVE DONE WELL.
YOU HAD HAD QUITE A BIT OF
TIME TO REFLECT THE EXPERIENCE
OVER THE YEARS SO IS THERE A
COMMUNITY OUT THERE THAT YOU
HAD TIME TO THINK ABOUT THIS
AND THEN TO SPEAK TO WHAT IS
THAT EXPERIENCE IS THERE A LOT
OF DISCUSSION ABOUT THIS HOW
IS THIS DISCUSSED BETWEEN
YOURSELF AND OTHERS.
>> ABSOLUTELY IT IS AN
EXCELLENT QUESTION I HAVE A
VERY CLOSE FRIEND I MET IN THE
TRIAL AND WHEN MY CHARGER
BROKE SHE LET ME BORROW HERS I
UNDERSTAND THEY CANNOT HAND
OUT INFORMATION FOR OTHER
PARTICIPANTS TO LET YOU BORROW
THEIR EQUIPMENT.
AND FOUR YEARS SHE HAD A
FACEBOOK GROUP FOR PEOPLE WHO
WERE CONSIDERING DEEP BRAIN
STIMULATION I WAS IN A
DOCUMENTARY PUT ON BY PBS AND
IN THE COURSE OF LIFE TREATING
THAT RELEASE I HAVE HAD PEOPLE
FROM BRAZIL AND OTHER
COUNTRIES REACH OUT TO ME ON
SOCIAL MEDIA AND SAY HEY I AM
FACING SIMILAR ISSUES WHAT ARE
THOSE THINGS I SHOULD BE AWARE
OF?
THERE IS A COMMUNITY AND
PLATFORMS THAT DISCUSS THIS
AND WHEN I WAS TALKING TO MY
FRIEND ABOUT POSTTRIAL WISH
LIST WE ALL HAVE SIMILAR
EXPERIENCES IN THESE
DISCUSSIONS DO HAPPEN THAT MY
FRIEND IS ONE OF THE PEOPLE
WHO THE INSURANCE COVERED IN
AND SAID WE WILL NOT COVER
THIS ANYMORE BECAUSE WE DON'T
COVER EXPERIMENTS.
WE ARE CHANGING A POLICY.
THOSE FEARS ARE FOUNDED BASED
ON THE EXPERIENCE OF PEOPLE I
INTERACT WITH I CAN SPEAK
PERIOD PUBLICLY WITH MY ISSUES
WITH DEPRESSION AND WHAT THE
TRIAL HAS DONE FOR ME AND THEY
DO THAT IN PART BECAUSE PEOPLE
CANNOT DISCUSS THIS BECAUSE
THERE IS STILL'S A LOT OF
STIGMA.
SO I SPEAK UP BECAUSE I CAN
AND NOT EVERYBODY ELSE CAN.
BACK IN THE DAYS THERE WAS A
GOOGLE PLUS I HAD AMA WHERE
PEOPLE JUST ASK QUESTIONS
ABOUT THE DEVICE AND THE TRIAL
AND MY EXPERIENCES AND
EVERYTHING FROM AN ENGINEER SO
HOW DO YOU RECHARGE THAT TO
WHAT IS ALL DAY BRAIN SURGERY
WHEN YOU ARE AWA AWAKE.
SO I NEEDED THAT WHEN WE FIRST
STARTED THE TRIAL.
YOU TALK TO THE OTHER PATIENTS
AND THAT SUPPORT WAS CRUCIAL.
I UNDERSTAND THAT'S NOT
NECESSARILY SOMETHING THE
TRIAL TEAM CAN COORDINATE BUT
IT ENDED UP TO BE CRITICAL FOR
MY LONG-TERM PARTICIPATION.
AND I WOULD NOT BE DOING AS
WELL I —- AS I AM IF I DID NOT
DEVELOP THOSE RELATIONSHIPS
WITH PEOPLE.
>> I HAVE SO MANY MORE
QUESTIONS I WILL PUT THEM IN
THE Q&A.
>> NOW WE WILL MOVE ON TO THE
NEXT PANELISTS.
THE ONE ISSUE THE QUESTIONS IN
THE Q&A THEY WILL NOT GO TO
THE PUBLIC RECORDING BUT WE
WILL DO OUR BEST TO RECYCLE
SOME OF THESE WITH THE PANEL
DISCUSSION.
>> SO NOW WE WILL MOVE ON TO
MISS JENNIFER FRENCH.
AS A RESULT OF THE
SNOWBOARDING ACCIDENT SHE
BECAME A QUADRA COLLEGIATE
FROM 1988 FROM C SIX AND SEVEN
SHE HAS A IMPLANTED NEURAL
PRODUCT THESIS SYSTEM SHE'S
FOUNDER OF THE EUROTECH
NETWORK A 501(C)3 NONPROFIT
ORGANIZATION THAT FOCUSES ON
EDUCATION AND SUPPORT FOR
NEUROTECHNOLOGY.
AND THEN THE AUTHOR OF BIONIC
PERIOD PIONEERS.
>> BRANDY, FANTASTIC JOB
YOU'RE A HARD ACT TO FOLLOW.
AND THAT GRATEFUL FOR THAT
IMPLICATION THE NEXT COUPLE OF
DAYS IT IS INCREDIBLY
IMPORTANT TO LOOK AT THE
EXPANSION OF THE IMPLANTED
DEVICES.
IF WE THINK OF THE DIFFERENT
ROLES THAT WE PLAY IN
NEUROTECHNOLOGY DEVICES THEY
CAN BE AND USERS AND TRIAL
PARTICIPANTS IN MAKING
DECISIONS THAT ALL OF THOSE
WITH LIVED EXPERIENCE WITH
INDIVIDUAL EXPERIENCES WITH
NEUROLOGICAL CONDITIONS.
AS A NONPROFIT AND THOSE
LIVING WITH NEUROLOGICAL
CONDITIONS.
NOT JUST THE FOUNDER BUT
SOMEBODY WITH THE CONDITIONS
OF 24 YEARS AGO I HAD AN
ACCIDENT THAT LEFT ME
QUADRIPLEGIC AND A YEAR AND A
HALF LATER I WAS IMPLANTED
WITH A NARROW PROSTHESIS AND
WITH LONG-TERM PARALYSIS I
REALIZE HAVING THIS DEVICE OF
HOW MUCH IT HAS IMPACTED MY
MEDICAL HEALTH AND MY ABILITY
TO COMBAT SECONDARY CONDITIONS
THAT THE ABILITY TO STAND
USING PARALYZED MUSCLES HAS A
LIFE IMPACT.
IT TO BE IN OTHER AREAS OF THE
BODY THOSE THAT ARE PUTTING
THEIR BODIES FORWARD WITH THE
ADVANCE OF NARROW TECHNOLOGY
AND THEN THE FIRST PARTICIPANT
TO USE THE BRAIN INTERFACE TO
INTERACT WITH PEOPLE WHO WAS A
LONG TIME TRIAL PARTICIPANT TO
REAP THE REWARDS AND WITH THAT
SPINAL CORD DEVICE LIVED WITH
CHRONIC PLANE AND NEVER FOUND
SOLUTION UNTIL SHE HAD THIS
DEVICE AND THEN THOSE WHO WAS
A PARTICIPANT TO RESTORE
SENSATION FOR A PROSTHETIC
LENS SO ALL OF THE'S PEOPLE
ARE JUST A FEW BUT WE PUT THEM
FORWARD AND RIGHTFULLY SO
THESE ARE THINGS THAT ARE
HAPPENING IN THIS FIELD THAT
WE TEND TO IGNORE AFTER THE
HEADLINES FADE IN THE TRIAL IS
OVER WITH AND THEN TO KEEP
THAT EXCITEMENT OF HOW PEOPLE
ARE LIVING.
AND OTHER LIVING WITH THAT
LONG-TERM AND THEN TO KEEP IN
MIND OF THE WHY AND I DRAW
YOUR ATTENTION TO THIS THE TOP
THREE REASONS OF WHY PEOPLE
PARTICIPATE IN CLINICAL TRIALS
AND IT IS COMPELLING TO KEEP
IN MIND AS WE MOVE TOWARD
SOLUTIONS IT IS A HORSE RACE
IT HAS POTENTIAL BENEFITS AND
THEN TO HELP ADVANCE SCIENCE
BUT ALSO ACCESS TO TREATMENTS
AND DEVICES LOOKING AT ALL THE
OTHER TREATMENTS THERE IS
NOTHING ELSE AVAILABLE FOR HER
IN A CLINICAL TRIAL IT WAS AN
OPTION FOR HER AND ALSO FOR ME
BUT LOOKING AT PERSONAL
BENEFIT'S WE HAVE TO SEE HOW
PEOPLE APPROACH CLINICAL
TRIALS AND WHY THEY
PARTICIPATE ONCE THE TRIAL IS
OVER WANT TO SHARE WITH YOU WE
RECENTLY PRESENTED THIS AT THE
INSTITUTES A SCIENTIFIC
RESEARCH AGENDA THAT THIS IS
JUST A PIECE OF THE FRAMEWORK
AS VALUE INTEGRATION WITH THE
MEDICAL WORKING GROUP AND THE
FACT THAT ETHICS NEED TO BE
EMBEDDED INTO THE SPECTRUM IS
NOT JUST THE INCEPTION AND
PLANNING STAGES BUT ENGAGEMENT
AND THOSE THAT CAN HELP PUT
SOME SCOPE IN PLANNING AND IN
THAT DEVELOPMENT STAGE THAT
KEY AREA OF TRANSPARENCY AND
ACCOUNTABILITY AND MOTIVATION
OF THOSE PLAYERS THAT ARE
ROOTED IN THAT DEVELOPMENT
STAGE AND THEN FINALLY
REGULATORY OVERSIGHT BUT ALSO
THE ETHICAL ISSUES THAT CENTER
AROUND CULTURAL AND SOCIETAL
ISSUES AND THAT COULD BE VERY
PRAGMATIC SO THAT IS A DRAFTED
FRAMEWORK TO GIVE YOU AN IDEA
SO WE HAD BEEN FOLLOWING
PEOPLE THAT ARE EARLY ADOPTERS
THERE ARE LOTS OF STORIES AND
DIFFERENT EXPERIENCES SO
LOOKING BACK AT THOSE STORIES
AND INTERVIEWS THAT HAVE BEEN
DONE IN PREPARATION WANT TO
SHARE WITH YOU THE EMERGING
THEMES SO ONE OF THE FIRST
AREAS WAS MAINTENANCE AND
REPAIR SO BEING ABLE TO LIVE
WITH THE DEVICE AND HOW YOU
GET IT REPAIRED IS A BIG
QUESTION BUT A LOT OF TIMES
ENGINEERS THINK OF BACKWARD
COMPATIBILITY BUT WHAT WILL
HAPPEN IN THE NEXT GENERATION
TO BE COMPATIBLE AND THEN TO
UPGRADE THE DEVICE LATER ON.
WHAT IS THE ELIGIBILITY?
AND THEN CONSUMABLES.
OF COURSE THERE ARE BATTERIES
BUT THERE ARE OTHER
CONSUMABLES THAT GO WITH
IMPLANTED DEVICES THAT COULD
BE EXTERNAL TO THE BODY AND
THE SECOND AREA OF
SIMILARITIES AND THEN THE
FINANCIAL BURDEN THAT COMES
ALONG HAVING NO LONG-TERM
IMPLANTED DEVICE SO THAT IS
OUT OF COSTUME AND THE FUNDING
ENDS IT GOES BACK TO THE
PARTICIPANT AND ALSO TO TRACK
THE USAGE OF THOSE DEVICES AND
IF THAT IS WORTHWHILE AND THAT
CONCERNED ABOUT LONG-TERM
FOLLOW-UP AND THEN HOW LONG
WILL IT BE FOR THE BENEFIT
ONCE THE TRIAL IS OVER?
>> THAT AREA OF EMERGING
ISSUES IS STILL ADVERSE EVENTS
AND LOSS OF FUNCTION.
AND THOSE OTHER ISSUES.
AND ALSO REFLECTING A LOSS OF
FUNCTION.
AND THEN HOW WILL THEY HAVE
LOST FUNCTION WHEN THEY PLANT
THE DEVICE AND THEN WHICH THE
PARTICIPANTS AND WHAT THAT
MIGHT ENTAIL AND WHAT THE
OPTIONS MIGHT BE.
IF THE TRIAL IS OVER WITH WHAT
ABOUT INFECTIONS WITH THAT
DEVICE AND THE PSYCHOLOGICAL
IMPACT AND PEOPLE THAT HAVE
BEEN IN VARIOUS TRIALS AND NOT
JUST NEUROPSYCHIATRIC BUT ALL
OF THE OTHER CONDITIONS OF
ONCE THE TRIAL IS OVER WITH
HOW DO WE HANDLE THE LOSS OF
FUNCTION OR THE LOSS OF
BENEFITS WE MAY HAVE ACHIEVED
DURING THE TRIAL?
>> AND FINALLY EXPECTATIONS TO
UNDERSTAND THE RISK AND WE
ALWAYS ASK THE QUESTION AND TO
UNDERSTAND THAT RISK EARLY ON.
BUT WITH THAT UNANTICIPATED
EXPECTATIONS ARE.
AND THAT IS PEPPERED WITH
LINGO ONE —- LEGAL LANGUAGE
AND WHAT THE PATHWAY COULD BE
IN THAT EXPECTATION AND THEN
TO RETAIN THE DEVICE WHAT THAT
MIGHT BE.
AND WITH THOSE IMPLANTED
DEVICES OF TODAY I WENT TO
HIGHLIGHT AND GO BACK TO GO
BACK TO LOSS OF FUNCTION.
AND A YEAR AND A HALF AFTER MY
INJURY TRULY THE INITIAL TRIAL
WAS 18 MONTHS THAT IS WHEN THE
TRIAL WAS OVER WITH.
IF I LOST THE ABILITY I COULD
NOT STAND OR USE ANY OF MY
PARALYZED MUSCLES.
BUT I WAS FORTUNATE ENOUGH TO
BE IN A TRIAL THAT WAS TO THE
CLEVELAND CENTER IN OHIO AND
IT ROLLED INTO A LONG-TERM
FOLLOW-UP STUDY AND I HAVE
BEEN THERE FOR THE LAST 23
YEARS.
OVER THOSE 23 YEARS THEY HAVE
FOLLOWING THE PROGRESSION AND
HOW I USE IT.
AND HOW PEOPLE USE IN SOCIETY
AND HOW IT IS USED IN SOCIETAL
I HAVE BEEN PARALYZED FOUR
TIMES OVER THE PAST FEW YEARS
THAT BECAUSE I AM INVOLVED IN
A LONG-TERM FOLLOW-UP STUDY I
CAN GET RE- IMPLANTED AND IN
SOME CASES UPGRADED TO THE
NEXT LEVEL OF DEVELOPMENT OF
DEVICES.
SO THAT LOSS OF FUNCTION IN
FACT IT HAPPENED VERY RECENTLY
DURING COVID MY SYSTEM FAILED
AND JUST LAST YEAR I WAS
REIMPLANTED SO IF YOU THINK OF
LONG-TERM STUDIES THAT IS
SOMETHING TO CONSIDER AND PUT
ON THE TABLE.
IS A MULTI AGENCY APPROACH.
THERE IS A WORKSHOP TO THROW
SOMETHING AGAINST THE WALL.
AND ALSO LOOKING AT THE FDA.
AND TO HAVE A MINIMAL PLAN IN
THE STUDY.
AND THEN HOW TO ADDRESS THEM
AND WITH THE INCOME TRACKING
AND THEN FUNDING MECHANISMS ON
THE NIH SIDE TO SUPPORT
LONG-TERM STUDIES WITH THE
ADDITIONAL FIVE YEAR RULE WITH
THESE PARTICIPANTS OVER A
PERIOD OF TIME AND
UNDERSTANDING THE EFFECTS BUT
ALSO THE DATA THAT IS PUBLICLY
AVAILABLE AND THERE'S AN
OPPORTUNITY FOR CMS WITH A
TRIAL PARTICIPANT IS A UNIQUE
OPPORTUNITY TO HAVE DATA
ANALYSIS FOR THOSE DECISIONS
THAT YOU CAN USE WITH PAYMENT
AND COVERAGE DECISION.
BUT IT GAINS AN UNDERSTANDING
OF WHAT AND WITH THOSE ADVERSE
ADVANCE MY HAPPEN IT IS JUST
ONE IDEA I WILL NOT CLAIM BUT
I KNOW THE SOLUTION.
AND THEN TO SPEAK WITH YOU AND
THOSE THAT WE RELY ON FOR
TECHNOLOGY.
AND ANY QUESTIONS THAT THEY
MAY HAVE.
>> THANK YOU SO MUCH WE CAN
ALLOW FOR ONE QUESTION.
IS THERE ONE QUESTION.
>> THANK YOU FOR THE
PRESENTATION I HAVE ONE
QUESTION TO FOLLOW UP ON WITH
THE VALUE OF FRAMEWORK
INTEGRATION PRESENTED AND WHAT
TYPE OF ENGAGEMENT YOU THINK
WILL BE MOST VALUABLE DURING
THE PROJECT OF THE INCEPTION
PHASE.
THANK YOU.
>> IT'S A GREAT QUESTION.
I'M A BIG ADVOCATE AND WITH
THAT EVEN IN THE EARLY PROCESS
AND IT WILL ALL DEPEND ON WHAT
YOU ARE DEVELOPING AND ALSO
UNDERSTAND WHAT THE TRIAL MAY
LOOK LIKE AND TO ENGAGEMENT
EARLY ON.
AND WITH THAT CLINICAL
PROCESSES IMPORTANT BECAUSE
AND IF WE HAVE SOMETHING
EXTREMELY IMPRACTICAL IT'S
DIFFICULT TO TRANSLATE THAT.
AND WITH A CONSUMER ADVISORY
BOARD AND THEN TO UNDERSTAND
THOSE PREFERENCES IN THE
PRIORITIES.
I HOPE THAT ANSWERS YOUR
QUESTION?
>> YES.
THANK YOU.
>> AGAIN, Q&A IS OPEN MOVING
ON TO THE THIRD SPEAKER FOR
THIS PANEL ASSISTANT PROFESSOR
AND WITH THE DEPARTMENT OF
PSYCHIATRY TO COMBINE HIS
BACKGROUND IN NEUROSCIENCE AND
BIOETHICS TO EXAMINE THE
IMPLICATIONS OF EMERGING BIO
MEDICAL TECHNOLOGIES IN
NEUROSCIENCE.
HIS CURRENT RESEARCH EXAMINES
WITH DEEP BRAIN STIMULATION
ASSISTANCE.
>> THANK YOU.
AND LOOKING AT POSTTRIAL
ACCESS.
AND THANK YOU BRANDY AND
JENNIFER FOR THEIR WONDERFUL
THOUGHTS.
YOU WILL SEE A LOT OF WHAT WE
ARE SEEING CURRENT TRIAL
PARTICIPANTS AND WITH THAT
EXPERIENCE THAT BOTH OF THEM
HAVE HAD.
WE HAVE DATA ON RESEARCHERS
WHERE THEY ASK ABOUT POSTTRIAL
ACCESS ISSUES.
AND THEN TO FOCUS ON THE
PATIENT PERSPECTIVE IN THE
PROCESS OF LOOKING FOR
JOURNALS.
>> AND WITH THAT DEEP BRAIN
STIMULATION AND THOSE
ASSOCIATED SYMPTOMS YOU TRY TO
MANAGE AND THEN TO IDENTIFY TO
BE ASSOCIATED WITH THE
SYMPTOMS AND THEN FOR THE
ONSET AND THEN ACTIVATING THAT
STIMULATION FOR THAT BRAIN
ACTIVITY AND FOR EPILEPSY BUT
THAT CONDITIONS WE ARE
DISCUSSING NO FDA APPROVAL.
SO WE INTERVIEWED THESE
PATIENTS BEFORE THE SURGERY.
WE ASKED THEM QUESTIONS ABOUT
POSTTRIAL DISCUSSIONS AND THEN
WE ASKED THEM SIX MONTHS LATER
WHAT TO DO REMEMBER?
AND THEY WERE PARTICIPATING IN
PARKINSON'S DISEASE TRIALS AND
TOURETTE SYNDROME.
SO WITH THE METHODS WE HAVE 21
PARTICIPANTS AND ALL
TRANSCRIPT WERE RECORDED BY
MEMBERS OF THE RESEARCH TEAM
AND WITH THOSE EXTRACTIONS AND
THEN TO EXTRACT THE THEME
COMING FROM THE DATA WE ALSO
HAVE AT LEAST TWO MEMBERS
LOOKING AT IDENTIFYING AND
THAT IS EMERGING SO WHAT IF
ANYTHING THROUGH THE TRIAL.
AND THE RESPONSES WERE NOT
VERY DETAILED.
SO WE LOWER THAT THRESHOLD SO
DO THEY HAVE ANY RECOLLECTION
OF HAVING DISCUSSED THIS OR
NOT?
AND AFTER THE CONSENT PROCESS
EIGHT OF THEM SAID THEY DID
NOT RECALL DISCUSSING THE
ISSUE EIGHT OF THEM DID SAY
THEY RECALL DISCUSSING THE
ISSUE AND FIVE OF THEM WERE
NOT VERY CLEAR SO ONE EXAMPLE
OF NO RECOLLECTION I DON'T
REMEMBER OR HAVING THAT
DISCUSSION.
SO NOW SIX MONTHS AFTER THE
SURGERY, POST SURGERY WE ASKED
AGAIN DO YOU REMEMBER HAVING
DISCUSSED THIS WITH THE TEAM?
SEVEN OF THEM SAID THEY STILL
DID NOT REMEMBER DISCUSSING.
'S YOU HAVE AN ISSUE HERE THE
UNETHICAL ISSUE OF CONSENT
WHERE MANY OF THE PARTICIPANTS
MAY BE UNDERGOING THE SURGERY
AND NOT NECESSARILY KNOW OR AT
LEAST IT WAS DISCUSSED IN THE
CONSENT PROCESS BUT THERE IS A
LOT GOING ON BUT THEY DON'T
RECALL HAVING DISCUSSED THE
ISSUES SO ONE EXAMPLE IS THAT
I CANNOT AFFORD THE NEW DEVICE
OR THE BATTERY OR WHAT IS
RECHARGEABLE ONE PARTICIPANT
IN THE SIX-MONTH POST SURGERY
INTERVIEWS SAID IT WAS FUNNY
BECAUSE AND THEN TO SAY THIS
QUESTION CAME UP ABOUT
BIOETHICS AND END OF TRIAL.
HOW DO YOU FEEL ABOUT ME IT'S
ON —- ASKING THAT QUESTION SO
THE PATIENT IS THEY ARE ABOUT
TO HAVE THE SURGERY AND THEN
REMEMBERS HE HAD THIS
DISCUSSION AND ONE OF THOSE
RESEARCH ASSISTANTS AND THEN
TO FEEL SOMEWHAT UNCOMFORTABLE
AT THAT MOMENT.
SO DO YOU THINK THEY SHOULD
WHAT SHOULD HAPPEN TO THE
DEVICE?
AND THEN 76 PERCENT SAID THEY
SHOULD KEEP THE DEVICE AND
HOPING IT WOULD PROVIDE AND
KEEPING IT.
AND THEY WERE NOT PROVIDE
THOSE ANSWERS AND WITH THAT
RESPONSE WAS 76 PERCENT FOR
MOST OF THEM AND THEY SAID WE
SHOULD BE ABOUT TO KEEP IT SO
81 PERCENT SAID THEY SHOULD BE
ABLE TO KEEP IT TOGETHER SAID
IT WAS UNCLEAR IF IT IS STILL
WORKING AND THEY HAVE TO FIGHT
ME FOR IT.
AND SO THEN THEY FEEL VERY
STRONGLY THEY WANT TO KEEP
USING THESE DEVICES AS IT WAS
BROUGHT UP THEY TRIED MANY
DIFFERENT TREATMENTS THAT ARE
FDA APPROVED BUT NOTHING HAS
WORKED WELL FOR THEM SO WHEN
THEY DO FIND SOMETHING THAT IS
WORKING THEY FEEL VERY
STRONGLY.
SO HOW DO YOU KEEPING
THE DEVICE FUNCTIONING AT THE
END OF THE STUDY SHOULD BE
PAID FOR?
AND INTERESTINGLY DURING THE
PRESURGERY INTERVIEW MOST
MENTIONED INSURANCE WHICH IS
REASONABLE.
FROM THEIR PERSPECTIVE ALSO
FOR RESEARCHERS IF IT IS
WORKING FOR THEM FOR A PERSON
THAT DOES NOT HAVE OTHER MID-
ON —- MEDICATIONS IF IT IS
WORKING FOR THEM THEN IT
BECOMES MEDICALLY NECESSARY
FOR THE INDIVIDUAL AND THEN
SHOULD ALLOW THIS FOR
INSURANCE PROVIDERS AND WITH
THE COST ASSOCIATED WITH THIS.
BECAUSE THIS COULD STILL BE
VERY EXPENSIVE FOR
PARTICIPANTS TO MANAGE THE
OTHER CALLS THAT ARE NOT
COVERED BY INSURANCE BUT SIX
MONTHS INTO THE SURGERY AND
THE DEVICE THAT MENTIONS
BASICALLY WHAT WE ARE
PRESENTING HERE AND THEN THEY
COULD MENTION MORE THAN ONE TO
HAVE SOME RESPONSIBILITY FOR
COVERING THE COST SO WE SEE A
DECREASE IN THE NUMBER OF
MENTIONS OF INSURANCE
PROVIDERS AND WHAT WE SEE IS
THERE IS AN INCREASE OF THE
NUMBER OF PEOPLE WITH DEVICE
MANUFACTURERS.
SO DURING THE PROCESS THEY
UNDERSTAND THE TRIAL PROCESS
AND THE DATA COLLECTED AND I
WILL MENTION SOME OF THE
ISSUES.
AND THEY WOULD SAY THEY HAVE
RESPONSIBILITY.
THEY MENTIONED THE RESEARCH
TEAM WHERE IT IS TAKING PLACE
AND SO FOR EXAMPLE THIS IS AN
EXAMPLE OF THE RATIONALE FOR
DEVICE MANUFACTURERS.
SINCE THE COMPANY OR THE TEAM
OR WHATEVER THEY DO HAVE A
RESPONSIBILITY TO CARE FOR
THEIR PEOPLE THAT HAVE THESE
DEVICES I WOULD NOT WANT TO BE
CUT LOOSE THERE ARE THINGS
HERE ABOUT RECIPROCITY AND
GETTING INFORMATION FROM THIS
RESEARCH AND THEN THE OTHER
PERSON THAT IS IMPORTANT TO
KEEP IN MIND SO ONE EXAMPLE IS
INSURANCE SHOULD PAY IS
SOMETHING THEY SHOULD HAVE TO
PAY FOR THE STUDY I THINK THEY
SHOULD TAKE SOME
RESPONSIBILITY AND IF THEY
JUST PAT WHAT THEY ARE
SUPPOSED TO DO THEN THEY SEE
THIS HAVING A LOT OF
EXPERIENCE TO GET INSURANCE TO
COVER THE DIFFERENT TREATMENTS
THAT THEY HAVE TRIED THAT HAVE
NOT WORKED FOR THEM BUT THIS
IS THE OBLIGATION WITH THE
INSURANCE PROVIDER SO THE
DEVICE HAS TO BECOME MEDICALLY
NECESSARY FOR THE PATIENT EVEN
THOUGH IT IS EXPERIMENTAL.
IF YOU CAN AFFORD IT THEN YOU
SHOULD PAY THE MAINTENANCE BUT
IF YOU CAN'T THE SAME WAY I
GOT INTO THE STUDY IS THE WAY
IT SHOULD BE.
BUT IT'S MORE THAN A FEW
PENNIES.
SO IT IS A FEAR THAT THE
COMPASSION THERE IS A MEDICAL
NEED THAT CANNOT BE MET
OTHERWISE SO EVEN IF THERE IS
A COST ASSOCIATED WITH THIS TO
FIGURE OUT A WAY TO MAKE IT
AVAILABLE IN CASE THEY CANNOT
AFFORD IT BUT ONE EXAMPLE AS
WELL THE STUDY SHOULD PAY FOR
IT THE PARTICIPANTS TOOK THE
RISK AND WHATEVER WAS NEEDED
TO BE PAID FOR BY THE
INDIVIDUAL PERSON IF THEY
CANNOT PAY THAN IT SHOULD BE
TAKEN CARE OF BY THE STUDY
EVEN WHEN THE STUDY ENDS ONE
EXAMPLE IS THERE IS A LIE THE
TIME AND EFFORT HAD TO GO
THROUGH BRAIN SURGERY I HAVE
TO COME IN EVERY TWO WEEKS AND
THAT TAKES UP THE WHOLE DAY
EVERY TWO WEEKS AND THEN EVERY
MONTH AND IT IS VERY TIME
CONSUMING SO WE SEE THAT
RECIPROCITY AND THEN
COMPASSION TO TAKE REASONABLE
STEPS TO MANAGE A NEED THAT
CANNOT BE MET OTHERWISE.
>> THANK YOU TO OUR
COLLABORATORS FROM THE BRAIN
RESEARCH.
>> WE HAVE TIME AGAIN FOR ONE
QUESTION.
>> THERE IS THE QUESTION IN
THE CHAT IF THE SUBJECTS IN
YOUR RESEARCH OR THE INDUSTRY
OR GOVERNMENT SPONSORED
STUDIES ARE BOTH?
>> MOSTLY GOVERNMENT-SPONSORED
BS MOSTLY GOVERNMENT.
>> NOW MOVING ON TO THE LAST
SPEAKER FOR THIS PANEL YOU CAN
CONTINUE TO ASK QUESTIONS FOR
THE PANEL DISCUSSION SO THE
LAST SPEAKER IS DOCTOR JAMES
MAHONEY AN ASSISTANT PROFESSOR
AND CLINICAL NEUROPSYCHOLOGIST
DEPARTMENT BEHAVIORAL MEDICINE
AND PSYCHIATRY AT WEST
VIRGINIA UNIVERSITY SCHOOL OF
MEDICINE.
HE ALSO SERVES AS DIRECTOR OF
INFECTIONS RESEARCH.
IS INTERESTING TO THE
INVESTIGATION OF NARROW
PSYCHOLOGICAL ISSUES WITH
SUBSTANCE USE DISORDER AS WELL
AS INVESTIGATIONS OF NOVEL
TREATMENTS INVOLVING
NEUROMODULATION WITH SUBSTANCE
DISORDER SUCH AS DEEP BRAIN
STIMULATION AND FOCUSED
ULTRASOUND AND MAGNETIC
STIMULATION.
>> THANK YOU FOR THAT
INTRODUCTION.
SO THANK YOU FOR THE
INVITATION TO SPEAK AND IT'S
GOOD TO SEE THERE IS A LOT OF
OVERLAP AND WHEN IT COMES TO
ENSURING THOSE NEEDS ARE MET
LOOKING IN THE DEEP BRAIN
STIMULATION FOR OPIOID USE
DISORDER SOMEONE WITH
ADDICTION AND SUBSTANCE ABUSE
DISORDER THE FIRST INVASIVE
TRIAL I WAS INVOLVED IN WE HAD
THIS PROPOSAL SO IT WAS VERY
WELL THOUGHT OUT.
AND WITH THAT MEDICATION
DEVELOPMENT THERE ISN'T AS
MUCH POSTTRIAL THEY ARE NOT AS
EXTENSIVE AS THE STUDY IT IS A
GOOD OPPORTUNITY WITH THE
SURGICAL TEAM ESPECIALLY.
AND THOSE WHO JOINED US HERE
IN WEST VIRGINIA UNIVERSITY
AND THOSE WHO HAVE BEEN DOING
THE DBS TRIALS.
BECAUSE THAT IS SOMETHING FROM
THE ONSET FOR WHEN THE STUDIES
ARE OVER, THEY ARE NOT OVER
AND THEN TO IMPACT DIFFERENT
METHODS TO ENSURE THE
PARTICIPANTS IN THE PATIENT'S
THAT THAT COMES FIRST.
SO SOME OF THOSE SPECIFIC ONES
REGARDING HARDWARE REPLACEMENT
IN THE FALSE GENERATOR
REPLACEMENT HAS TO OCCUR.
THE CURRENT STUDY I WAS
DISCUSSING HAS A 52 WEEK
PRIMARY COMPONENT AND THEN
WITH AN EXTENDED FOLLOW-UP
OVER FIVE YEARS.
SO THAT GIVES US THE ABILITY
BUT CLINICALLY BUT JUST
RESEARCH PURPOSES AS WELL IN
MY THAT'S IMPORTANT.
AND DEVICE MAINTENANCE TO
FOLLOW THE'S INDIVIDUALS FROM
WHEN THE PROTOCOL AND JUST
MAKE SURE THE DEVICE IS
WORKING PROPERLY AND THEN ALSO
SIDE EFFECT MANAGEMENT THAT
DOESN'T AND WHEN THE STUDY
ENDS.
C HAVE TO CONTINUE TO KNOW THE
SAFETY IS AT THE FOREFRONT.
LOOKING AT THE
RESPONSIBILITIES ASSUMED BY
THE INVESTIGATIVE TEAM THEY
NEED TO BE CONSIDERED VERY
EARLY ON FROM THAT PROPOSAL IN
THE PROTOCOLS DEVELOPED HOW DO
YOU SET FUNDING SOURCES THESE
ARE DISCUSSED IN THE
APPLICATION PROCESS AND HOW
WILL THE POST CARE TRIAL
COMPLETION AND THEN THE
PARTICIPANT WITH THE CONSENT
PROCESS TO MAKE SURE IT IS AS
INFORMED AS POSSIBLE WORKING
WITH INDIVIDUALS WITH
ADDICTION BECAUSE THERE IS
ANXIETY AROUND DAY TODAY AND
THE CLINIC LET ALONE THE
UNCERTAINTY FOR WHEN THE TRIAL
IS OVER HOW DO I MAKE SURE I
AM TAKEN CARE OF AND THEN TO
DISCUSS WITH THE FOLLOW-UP
CARE WILL LOOK LIKE AND GOING
FORWARD WITH CONSENTING AND TO
BE COMPLETELY TRANSPARENT AND
THEN THE INVESTIGATIVE TEAM
THAT CAN HELP YOU BRIDGE THE
COST AND THEN THE ULTIMATE
GOAL MOVES TOWARDS FDA
APPROVAL AND THEN HOPEFULLY IT
COVERS SOME OF THOSE COSTS BUT
WHAT CAN BE DONE IN THE
MEANTIME BEFORE THAT HAPPENS?
SO IT'S NOT JUST THE
INVESTIGATOR IT IS A
COLLABORATIVE EFFORT
UNIVERSITY OF HEALTHCARE
INSTITUTIONS WITH THOSE
INSURANCE PROVIDERS AND
INDUSTRY PARTNERS AS WELL
FUNDING THE AGENCIES THROUGH
PRIVATE DONORS WHEN THE
INVESTIGATIVE TEAM IS
DEVELOPING PLANS OF HOW THEY
WILL BE FINANCIALLY SUPPORTED
YOU ARE LOOKING AT ALL THE
OPTIONS.
AND THEN TO MAKE SURE WITH THE
FINANCIAL BURDEN WHETHER
ADDITIONAL STAFFING NEEDS AND
TO MAKE SURE THOSE NEEDS ARE
MET.
ALSO SEPARATING OUT CLINICAL
NEED AND FOR THOSE DEVICE
TRIALS WITH THAT DEEP BRAIN
STIMULATION THAT DBS WILL
REPLACE ALL OF THEIR OTHER
CLINICAL TREATMENT.
WE DON'T THINK THAT THAT IT
WILL JUST FLIP THE ADDICTION
SWITCH AND NOW THEY NO LONGER
NEED THERE BE.
LOOKING AT IT AS A CONJUNCTIVE
TREATMENT THIS IS JUST ANOTHER
TOOL IN THE TOOLBOX WITH THE
STANDARD LEVEL OF CARE.
BUT IS JUST MEANT TO AUGMENT
THE TREATMENT THEY ARE ALREADY
GETTING AND THEN ALSO AND THEN
THOSE INVESTIGATORS AND THEN
TO RELOCATE AND RETIRE.
AND THEN TO ENSURE THAT
INDIVIDUAL CAN CONTINUE.
TALKING SPECIFICALLY WITH THAT
DEEP BRAIN STIMULATION
PROTOCOL WE SAT DOWN AND ARE
WRITING UP THE INITIAL
PROPOSAL AND WE ARE DEFINITELY
CONSIDERING WHAT WILL BE DONE
WITH THAT ONE YEAR FOLLOW-UP
PHASE AND LOOKING AT THE
LONG-TERM FOLLOW-UP WE HAVE
EXTENDED THE FOLLOW-UP TO FIVE
YEARS AND ONCE WE GET TO THAT
POINT HOPEFULLY EVEN LONGER
AND THEN WITH DBS THEY PARTNER
WITH THEM AND ALSO
INSTITUTIONAL SUPPORT WITH THE
ADMINISTRATION FOR THE
COVERAGE AFTER THE TRIAL IS
COMPLETED AND THEN ALWAYS
HAVING INCLUSION OF OTHER
INVESTIGATORS WHO HAVE TRADING
AND EXPERTISE AND THAT IF IT
IS NO LONGER AVAILABLE WE HAVE
CONSIDER THESE FACTORS WITH
THAT PROTOCOL THAT WE HAD OF
OUTPATIENT MONITORING THAT WE
EXTEND THAT AND THEN TO LOOK
INTO OTHER SUPPLEMENTARY
SUPPORT TO COVER THOSE COSTS
AND OTHER FINANCIAL
CONSIDERATIONS AND THEN
LOOKING INTO SECONDARY AND
EXPLORATORY AND FROM THE ONSET
AND AS A TRIAL PROGRESSES
BASED OFF ON THE PATIENTS
RESPONSES THAT IS A PLANET - -
PRELIMINARY ANALYSIS TO SEE IF
THERE WAS SUPPLEMENTARY
FUNDING AND TO COVER THOSE
HARDWARE COST WITH THE GOAL OF
MOVING TOWARDS FDA APPROVAL IN
THE MEANTIME.
SO SPECIFIC TO US WE HAVE A
PROPOSAL FROM 2018 THE
NEUROSTIMULATOR FROM THE
ACTIVA PC THERE WAS A NEW
DEVICE AND THAT CAPABILITY OF
THOSE POTENTIALS THAT GAVE US
ANOTHER DATA POINT THAT DIDN'T
INCLUDE THE ORIGINAL PROPOSAL
TO GIVE US THE OPPORTUNITY
AFTER HE SUBMITTED THE
AMENDMENT WITH THE FDA
REQUESTING THAT DEVICE AND NOW
WE COULD GET PRELIMINARY DATA
TO POTENTIALLY WRITE
SUPPLEMENTARY GRANTS AND
PROPOSALS TO USE THE SAID
ADDITIONAL DATA CAPTURING
MECHANISM AND CONTINUOUS
LONGER-TERM CARE PAST THE FIVE
YEARS.
SO DEFINITELY IT'S ONE WAY TO
LOOK AT SUPPLEMENTARY FUNDING
TO CARRY OVER FOR FDA APPROVAL
AND UNTIL THAT IS SECURED.
SO IN SUMMARY THE
INVESTIGATIVE TEAM THAT ALSO
IN COLLABORATION WITH OTHERS
THAT SHOULD BE CONSIDERED VERY
EARLY ON YOU DON'T WANT TO
WAIT UNTIL THE END OF THE
STUDY WHAT WE DO WITH
POSTTRIAL CARE AND BEFORE THAT
IS SUBMITTED VERY EARLY ON
ALWAYS HAVE TO KEEP THE
PARTICIPANTS AT THE FOREFRONT.
THAT TRANSPARENCY WITH THE
PARTICIPANTS WHEN DISCUSSING
POST TRIAL CARE WHAT IS
EXPECTED OF THEM AND OF THE
TEAM AND WHAT METHODS ARE PUT
INTO PLACE TO MAKE SURE THEIR
CARE WILL BE MET WITH THE POST
STUDY COMPLETION AND THEN ALSO
LOOKING INTO THOSE OTHER
FUNDING MECHANISMS TO MAKE
SURE THOSE POSTTRIAL NEEDS ARE
MET AND CONTINUOUSLY
REVISITING THAT AND THEN ALSO
LOOKING AT THE OTHER OPTIONS
AND THEN I'LL ANSWER ANY
QUESTIONS.
>> WITH THE QUESTION
SPECIFICALLY FOR THIS
PRESENTATION ANY QUESTION
SPECIFICALLY FOR DOCTOR
MAHONEY?
>> THIS IS A FASCINATING
STUDY.
I SEE SUCH SIMILARITIES WITH
HER TALK AND THINKING ABOUT
THE AGE OF WHAT IS LIKELY IN
THE PREDICTION STUDY AND I AM
JUST WONDERING IT SEEMS
FANTASTIC IN SOME WAYS HE WILL
BE THAT I JUST WONDER THOSE
PEOPLE THAT WILL HAVE 30 PLUS
YEARS WITH A DEVICE IF IT IS
WORKING FOR THEM.
IS THE UNIVERSITY OFFERING
HAVE THEY TALKED ABOUT A TIME
LIMIT?
HOW HAS THAT BEEN HANDLED
QUICK.
>> ABSOLUTELY NOT AND FROM THE
BEGINNING THIS IS THE ANALYSIS
WITH A VERY HIGH RISK OF DEAF
AND THEY HAVE TO HAVE MULTIPLE
OVERDOSES SO THAT THE HIGH
RISK OF DEAF BUT I BROUGHT
THAT UP IN THE BEGINNING SO
WITH 30 YEARS OF AGE THERE IS
NO REASON BUT THEN EVERY
COUPLE OF YEARS WITH THOSE
BATTERY REPLACEMENTS AND IN
THE ONGOING SUPPORT SO THAT
FIVE YEARS AND THAT ATTENTION
IS FURTHER ON FOR THE FIRST
TEN OR 15 YEARS SO WE ARE VERY
FORTUNATE TO HAVE THEIR
SUPPORT AND WHAT'S GOING ON
RIGHT NOW ESPECIALLY IN WEST
VIRGINIA IN TERMS OF THE
ADDICTION CRISIS WE DO HAVE
THAT SUPPORT HERE BUT IT IS A
GREAT POINT WITH THE
RECHARGEABLE ONES BUT WITH
THIS INITIAL STUDY GIVEN ALL
THE CHAOS FROM THE SUBSTANCE
USE DISORDER RELYING ON HOW TO
RECHARGE IT AND ALL OF THAT
BUT THEN SOMETHING ME WILL
EXPLORE IN THE FUTURE
DEFINITELY BUT YOU ARE RIGHT
THAT'S WHY IT'S GREAT TO HAVE
WORKSHOPS LIKE THIS BECAUSE
THIS NEEDS TO BE DISCUSSED AND
CONSIDERED EARLY ON IN THE
PROCESS BECAUSE WE ARE
RECRUITING INDIVIDUALS WITH A
HIGH RISK OF DEAF BUT
POTENTIALLY WE ARE GIVING THEM
THEIR LIVES BACK.
THAT'S THE CASE WE NEED TO
ENSURE WE TAKE CARE OF THEM
CONTINUOUSLY.
>> SO WHAT MISS ALICE OR
MS. FRENCH HAD AND THEN WE HAD
SOME EXPERIENCE WITH PEOPLE
WHO HAD DEVICES OVER DECADES
SO WE ARE CURIOUS TO HEAR
ABOUT THAT EXPERIENCE AND THEN
TO INVITE THE OTHER PANELIST
TO TURN ON THEIR CAMERAS I AM
A NEUROLOGIST AND THE DIRECTOR
OF BIOETHICS.
>> I AM A SPECIALIST WORKING
WITH THE BRAIN NEWG.
>> SAID TO ANSWER THE QUESTION
WE WOULD LIKE TO HEAR A LITTLE
MORE ABOUT HOW FAR INTO THE
FUTURE THEWERE THINKING ATTHE TD
WAS 18 MONTHS ANOTHER MAY HAVE
BEEN LESS CLEAR BUT WHAT KIND
OF CONVERSATIONS DID WE HAVE
WHAT CONVERSATIONS SHOULD WE
BE HAVING WHERE THE HORIZON IS
VERY MURKY?
>> BEFORE WE BEGIN TO ANSWER
THE QUESTION I WANT TO WELCOME
TYLER WHO IS A PATIENT
PARTICIPANT HE CAN JOIN
TODAY'S WE WELCOME YOUR INPUT
AS WELL AS PERSPECTIVES AS
PART OF THE PANEL.
>> WITH MY ORIGINAL TRIAL I
WENT TO THE ATLANTA AREA I HAD
TO AGREE TO BE FOLLOWED UP FOR
AT LEAST ONE YEAR AND AFTER
THAT IT NATIONALLY
TRANSITIONED AND THE
DISCUSSIONS ORIGINALLY WERE
ALONG THE LINES OF TEN YEARS
OR FDA APPROVAL WHICHEVER
COMES FIRST.
THE TEN YEARS CAME FIRST I
UNDERSTAND THAT IS A FINITE
NUMBER BECAUSE A PARTICIPANT
IN THE EXPERIMENTAL TRIAL FOR
THE REST OF MY LIFE WITH NO
DEFINED TERMS.
IT'S MY UNDERSTANDING IS
SOMETHING CHANGES I NEED IT
REPLACED OR THERE'S SOMETHING
WITH THE IMPLANT I NEED TO BE
WE CONSENTED.
BUT THE TRANSITION FROM YOUR
NUMBER ONE THROUGH TWO OR
THREE OR FOUR WAS NOT A BIG
CHANGE.
MY TRIAL TEAM HAS BEEN VERY
COMMUNICATIVE FOR AS LONG AS
I'M WILLING TO DO FOLLOW-UPS
, THEY WOULD LIKE THAT.
AND I'M TRYING TO HELP IN ANY
WAY THAT I CAN.
BUT THERE WERE ALLIED OF
QUESTIONS ABOUT WHAT HAPPENS
AFTER TEN YEARS?
AND IT IS FRUSTRATING THAT
NOBODY KNOWS ANYTHING MORE
THAN I DO NOBODY KNOWS WERE
THIS WILL BE TEN OR 20 OR 30
YEARS DOWN THE ROAD AND WHAT
MY OPTIONS WILL BE OR WHERE I
MIGHT HAVE SURGERY IF I NEED
ANOTHER SURGERY.
THOSE ARE KNOWN UNKNOWNS AND
THEY DO CAUSE ANXIETY FOR
PARTICIPANTS BUT I DON'T KNOW
IF IT IS REASONABLE TO EXPECT
MY TRIAL TEAM TO KNOW HOW I
WILL BE TREATED 20 YEARS FROM
NOW.
BUT THIS PAST NOVEMBER WAS
MADE TEN YEARS I AM NOW
GRADUATED.
HOW TO PHRASE THAT BUT I AM
NOT UNDER CONSENT ANYMORE.
>> IT'S INTERESTING BRANDY
COME I HAD A SIMILAR
EXPERIENCE.
MY NOW HUSBAND WE MOVED TO
CLEVELAND TO PARTICIPATE SO
ONLY THOSE THAT ARE FLUENT IN
THE SUPPORT SYSTEM AROUND THEM
WE HAVE TO THINK OF THOSE
FINANCIAL BURDENS FOR
PARTICIPATION BECAUSE IT IS A
HUGE PERIOD FOR A LOT OF CAN
CLINICAL TRIAL PARTICIPANTS
AND FOR ME PERSONALLY I WAS
INJURED 24 YEARS AGO AND I HAD
NO IDEA I WOULD BE INJURED
THIS LONG OR THAT THE DEVICE
WOULD CONTINUE TO WORK THIS
LONG MANY INVESTIGATORS ARE
STILL AMAZED ONE OF MY
ORIGINAL IPG IS STILL TAKING
KNOCK ON WOOD.
BUT THAT ALLOWS FOR FUNCTION
AND THEN TO APPLY THE
FORESIGHT WITH THE RESEARCH
AND DEVELOPMENT PORTFOLIO THEY
TOOK A DISTINCT INTEREST TO
FOLLOW LONG-TERM TO UNDERSTAND
WHAT THEY ARE OF THOSE DEVICES
AND WHEN WE THINK ABOUT VERY
EXPERIMENTAL DEVICES LONG-TERM
STUDIES CAN HELP US FURTHER
DEVELOP THAT TECHNOLOGY AS WE
GO ALONG SO IT'S IN OUR BEST
INTEREST HOW WE PUT THESE
DEVICES AT WORK AND ALSO WITH
THE CLINICAL TRIAL
PARTICIPANTS.
>> AND THEN TO SEE IF WE WANT
TO WEIGH IN AS WELL?
>> I AM IN THE SAME STUDY AS
BRANDY.
I'M JUST TRYING TO THINK WHEN
I FIRST HAD THE SURGERY IF IT
WAS THAT BUT THERE WERE NOT
MANY OPTIONS.
WASN'T THINKING ABOUT THE
FUTURE TOO MUCH AND NOW I'M
THREE YEARS INTO THIS AND NOW
WITH YOUR NUMBER THREE IN MY
PART OF THE TRIAL WAS OVER
MAYBE ONLY FOR SIX MONTHS THE
DOCTOR THAT I COMMUNICATED
WITH IT WAS THE ACTUAL NUMBER
BUT I WASN'T THINKING ABOUT
ANY OF THESE THINGS BUT IN MY
BATTERY WAS RECHARGEABLE
MUSCLES TO LAST 15 YEARS BUT
AGAIN BEFORE BRANDY SHARED AND
NEVER CROSSED MY MIND THAT IT
COULD MALFUNCTION OR IF IT DID
I HAVE TO LIVE DAY BY DAY OR
MY EXAM ZAIDI WILL GET OUT OF
CONTROL SO I CROSS THOSE
BRIDGES WHEN I COME TO IT AND
IT WOULD BE GREAT TO HAVE
THOSE THINGS THAT RANDY
MENTIONED AND THAT WOULD BE
INCREDIBLE BECAUSE THE PAST
YEAR AND THEN I DO HAVE
INSURANCE AT THIS POINT AND IT
DOES CARE FOR THOSE
MAINTENANCE PROCEDURES BUT WHO
KNOWS WHAT THE FUTURE WILL
HOLD THAT NOBODY KNOWS WHAT
WILL HAPPEN TOMORROW WITH B OR
C OR D BECAUSE OF THIS DOESN'T
WORK I DON'T HAVE A LIFE.
I'M 31 YEARS OLD AND I HAVE
ALREADY LAST TEN YEARS TO A
TREATMENT RESISTANT DEPRESSION
AND I WOULD LIKE TO START
PICKING UP THE PACE A LITTLE
BIT AND AGAIN HAVING JENNIFER
HERE AS WELL 24 YEARS OF THIS
EXPERIENCES EYE-OPENING AS
WELL SO I AGREE WITH ALL THE
POINTS THAT THEY MADE AS FAR
AS PLAN OF ATTACK AND THE
GUARANTEED COVERAGE IS HELPFUL
AND WE ARE NOT MADE OF MONEY
PER SE BUT WE HAVE BEEN ABLE
TO FIND WAYS TO PURSUE THIS
EDGES LIFE OR DEAF OF FIVE TO
ACCUMULATE DEBT TO STAY ALIVE
THAN IT'S WORTH IT FOR ME
BECAUSE THEN I HAVE THE
OPPORTUNITY TO PAY OFF THE
DEBT.
BUT IT WOULD BE GREAT TO GET
HELP WITH AS WE HAVE TO KEEP
BUILDING THAT UP.
THANK YOU.
>> SO FURTHER ALONG IN THE
COMMERCIALIZATION OF THE
DEVICE SOME OF THOSE MAY
REMEMBER MORE RECENTLY THE
SECOND SITE AND WITH THAT
DEVELOPMENT OF THOSE DEVICES
VERY EARLY ON TO BRING
PROFOUND IMPACT AND TO THINK
WHAT HAPPENS WHEN A COMPANY
GOES OUT OF BUSINESS ARE NO
LONGER AVAILABLE AND HOW
SOMEONE TRANSITIONS AWAY FROM
THAT AND WHAT THAT MIGHT LOOK
LIKE THERE WAS A REALLY GOOD
COMMENTS IN THE CHAT IF
SOMEONE IS GOING TO RECEIVE
COVERAGE THEY WOULD BE A
LONG-TERM TRIAL PARTICIPANTS
SO IT IS A TWO WAY STREET
THOSE WHO ARE A PARTNERSHIP OR
A COLLABORATION WE ARE TRY TO
GET TO THE POINT AND TO
ENCOUNTER HEALTHWISE.
>> THESE VOICES ARE REALLY
POWERFUL AND HEARING YOU ALL
ARE ALL VERY GRATEFUL AND WITH
THE TOPICS WITH A Q&A WITH THE
PANEL DISCUSSION AS WELL SO TO
HAVE THE OPPORTUNITY TO INJECT
SOME THOUGHTS INTO THE
DISCUSSION.
>> FIRST OF ALL THANK YOU THE
PANEL WAS WONDERFUL AND FOOD
FOR THOUGHT.
WITH THE REAFFIRMED TO THE
NEEDS AND THAT'S WHAT I
UNDERSTAND ARE INCREDIBLY
IMPORTANT BUT I HAVE HEARD TO
THE HAVE NOT HEARD BEFORE SO
ONE WAS THAT JENNIFER
MENTIONED REGISTRY OR PUBLIC
DATA AND I WANT TO UNDERSTAND
HOW THAT IS MEETING ONE OF
YOUR NEEDS?
ONE OF THE REASONS THAT IS
HELPFUL AND SIMILARLY I THINK
IT IS BRANDY THAT MENTIONED
INCENTIVES TO MAINTAIN
COMPATIBILITY.
HOW DOES THAT HELP IN TERMS OF
LONG-TERM NEEDS?
THANK YOU.
>> YES.
I'M HAPPY TO SPEAK TO MY POINT
FOR ME THE REASON I MENTIONED
REGISTRY IS THAT I AM
CONTEMPLATING IF I DECIDE TO
RETIRE TO PANAMA AND MY TRIAL
TEAM IS ALSO RETIRED IS HAVING
A CENTRAL DATABASE THAT SAYS
THIS PERSON WAS LEGITIMATELY
INVOLV INVOLVED.
HERE IS THERE SETTINGS AND
HERE IS WHERE THEY CAN GET A
DEVICE.
AND THAT SORT OF INFORMATION
THAT CAN BE, LIKE MY MEDICAL
RECORD DOESN'T ACTUALLY
CONTAIN THE TRIAL.
IT CONTAINS MY SURGERIES BUT
IT DOESN'T HAVE MY TRIAL
INFORMATION.
SO I WORRY ABOUT BEING
SOMEWHERE ELSE AND BEING ABLE
TO PROVIDE DOCUMENTATION WHAT
THE SETTINGS ARE SUPPOSED TO
BE WITH THAT INFORMATION
BECAUSE I CANNOT CHANGE MY
SETTINGS.
TO HAVE ANY IMPACT SO IN THE
ABSENCE IF ATLANTA IS NOT
ACCESSIBLE FOR THERE TO BE A
WAY TO GET THAT INFORMATION
AND WITH BUT I CONSENTED TO I
KNOW I CAN ASK FOR THEM BUT AT
THE SAME TIME IT'S BEEN TEN
YEARS BUT FOR INCENTIVES, IT
IS TWOFOLD FOR THOSE THAT ARE
UNDERREPRESENTED TO GET THE
OPPORTUNITY TO PARTICIPATE IN
THESE TRIALS.
BECAUSE WE NEED THAT.
BUT WHEN RATINGS TAKE ALL
DAY, IF YOU ARE CAPABLE OF
WORKING THEN YOU NEED MORE
THAN JUST YOUR PARKING
VALIDATED IT IS SIMILAR TO
JURY DUTY IF NOBODY DOES IT
FOR THE MONEY AND THERE NEEDS
TO BE A WAY TO MAKE IT
POSSIBLE FOR PEOPLE WITH
DIFFERENT MEANS AND
UNDERREPRESENTED TO HAVE
ASSISTANCE WITH RELOCATION
WERE TAKING TIME OFF OF WORK
AND SEPARATE FROM THAT I WOULD
LOVE FOR MY DEVICE
MANUFACTURER TO CREATE AND
HARNESS THAT WHICH ALLOWS MY
ELECTRODES TO BE SWAPPED OUT
WITH ANY BATTERY ON THE MARKET
BUT WHY WOULD THEY DO THAT IF
THEY DON'T HAVE AN INCENTIVE
TO STANDARDIZE THE THINGS THEY
ARE IMPLANTING?
AND TO MAKE THEM CROSS
COMPATIBLE.
I WANT THEM TO HAVE A REASON
WHY MY DEVICE CAN BE SWAPPED
OUT BECAUSE I MOVE SOMEWHERE
ELSE AND DON'T HAVE ACCESS TO
THIS PARTICULAR DEVICE
MANUFACTURER ANYMORE.
SO WITH WANTING INSURANCE
COVERAGE AND COMPATIBILITY AND
I UNDERSTAND THAT BUSINESSES
THAT ARE INVOLVED IN THIS ARE
NOT NECESSARILY DOING THIS OUT
OF THE KINDNESS OF THEIR HEART
THAT TO HAVE A REASON TO MAKE
THESE TRIALS SUCCESSFUL
LONG-TERM AND TO HAVE THEM BE
ABLE TO COUNT ON IF THEY
CREATE A DEVICE THAT THEY WILL
NOT LOSE OUT ON WHATEVER
BUSINESS.
SO THAT WAS THAT YOU NEED THE
DEVICE PEOPLE TO STICK AROUND
YOU NEED PARTICIPANTS TO BE
REPRESENTED AND YOU NEED
OUTSIDE SOURCES TO GET ALL OF
THE DATA THAT THEY NEED AND
INCENTIVES ARE REALLY THE ONLY
LANGUAGE THAT I KNOW OF HOW TO
GET THAT COMMUNICATED.
I THAT HELPS TO CLARIFY.
>> I WOULD LIKE TO TAKE A FEW
SECONDS TO ADD ON.
WHEN YOU LOOK AT INCENTIVES
WHEN WE ARE DESIGNING CLINICAL
TRIALS IS MAPPING OUT THE
PATIENT JOURNEY INTO HAVE AN
IDEA WHERE THE PAIN POINTS ARE
AND WHAT ARE THEIR MOTIVATIONS
FOR JOINING THE TRIALS THAT IS
THE INCENTIVE NOT NECESSARILY
THE FINE LINE OF PAYMENT BUT A
LOT OF INCENTIVES AND
DIFFERENT FORMATS AND THEN
GOING INTO PUBLICATIONS IT'S
IMPORTANT NOT TO PUBLISH ONLY
NOT WHAT WENT WRONG BUT WHAT
WENT RIGHT SO WHAT YOU ARE
DEVELOPING IS ALIGNING TO BE
ADDRESSED AMONG THE POPULATION
SO PUBLISHING THAT DATA IS
INCREDIBLY IMPORTANT NOT JUST
FOR THE SCIENTIFIC COMMUNITY
BUT THE LAY COMMUNITY AS WELL
BECAUSE TO TAKE US TO THE
PRIMARY CARE NOT ALWAYS UP ON
THE RESEARCH OF CONDITIONS IS
SOMETHING WE CAN BRING FORWARD
TO IT SCIENCE IS DOING TO HAVE
AN INFORMED CONVERSATION WITH
YOUR CLINICIAN AND THEN
FINALLY AN INCREDIBLY
IMPORTANT POINT TO EVEN WITH
THE PROSTHETIC STUDIES AND
YEARS AGO TO GET THEIR
PERSPECTIVE THAT THEY WERE
JOINING THE REGISTRY
VOLUNTARILY THEY COULD DO SO
WITH BACKGROUND INFORMATION TO
REALLY UNDERSTAND WHAT WENT
WRONG AND WHAT WENT WELL SO WE
CAN LEARN FROM PREVIOUS TRIALS.
>> WE ARE AT TIME THAT I WAS
HOPING WE COULD EXTEND
DISCUSSION FOR ANOTHER TEN
MINUTES BECAUSE THE
PRESENTATIONS RAN LONG.
WE STILL HAVE A 35 MINUTES OF
BREAK I AM HOPING WE CAN
CONTINUE THIS DISCUSSION A
LITTLE LONGER.
>> SO ONE QUESTION I WANTED TO
POST TO THE PANELIST IS THAT
INFORMATION GATHERING DIALOGUE
ONE OF THE NEEDS THAT WAS
RAISED WAS RELATED TO DEVICE
EXPECTATION OR TO THE MEDICAL
INDICATIONS SO WE WANTED TO
GET THE PANELIST THOUGHTS IF
THAT RESONATES AND THE
THOUGHTS AROUND THAT
PARTICULAR MISTRIAL NEED.
>> SO WERE HAPPY TO JUMP IN TO
THE PARTICIPANTS OF THE
STUDIES SO ONE ISSUE IS WHEN
THEY ARE GOING IN I'M HOPEFUL
THAT THIS WILL WORK FIRST ON
WILL SAY I DON'T KNOW IF IT'S
GOING TO WORK BUT THEY'RE NOT
THINKING OF HAVING THE DEVICE
REMOVED AND I HAVE SEEN SOME
DISCUSSIONS SO THIS CREATES AN
INTERESTING RELATIONSHIP AND
THAT YOU WILL BE IN THIS
PATIENT'S BRAIN FOR THE
DURATION OF THEIR LIFETIME SO
TO CREATE A LINK PLUS WITH THE
DEVICE TO CREATE A CONNECTION
THAT WILL LAST A LIFETIME AND
THE LIFETIME OF THE PATIENT.
SO I HAVE SEEN SOME PEOPLE SAY
THEN WE SHOULD REMOVE THE
DEVICE OR OFFER TO REMOVE THE
DEVICE WHICH OBVIOUSLY THEY
HAVE TO PROVIDE CONSENT BUT
THERE ARE CONCERNS ABOUT THE
SURGERY AND WHEN PATIENTS DO
WANT THE DEVICE REMOVED AND IF
IT IS SAFE TO REMOVE THE
DEVICE THERE IS A NUMBER OF
CONSIDERATIONS.
BUT WE ONLY HEARD OF ONE GROUP
THAT IS ACTUALLY REQUESTING
FUNDS TO NIH TO HAVE ONE OR
TWO DEVICE REMOVALS.
PEOPLE ARE THINKING ABOUT IT
AND IT IS POSSIBLE TO REQUEST
THOSE FUNDS AS I UNDERSTAND
, BUT IT'S NOT SOMETHING THAT
PEOPLE ARE OFTEN DOING BECAUSE
THAT THEN IN THAT SENSE THAT
THEY MAY NEED THOSE FUNDS.
>> THAT IS A GREAT POINT.
IT CAME UP DURING THE PRE-
INTERVIEWS IN PREPARATION FOR
THIS MEETING WITH THE BRAIN
COMPUTER INTERFACE
PARTICIPANTS BUT MAYBE THEY
WERE NOT READY AT THAT TIME.
AND THEN THE FUNDS RUN OUT.
AND THEN PUTTING ASIDE FUNDS
FOR THE INDIVIDUAL.
AND THEN CONCERNS TO HAVE A
VERY QUICK DECISION EVEN
THOUGH IT MAY HAVE BEEN
BROUGHT FORWARD HOW DOES
SOMEBODY PREPARE FOR THAT AND
BE EMPATHETIC AND WHAT IS THE
DECISION TREE OR THE
CONTINGENCY FOR A TRIAL
PARTICIPANT?
>> AND WITH THOSE SOLUTIONS
THAT ARE OFFERED NOW AND TO
MAKE THE POINT THAT ONLY THE
LATER PANELS AND MAKING USE OF
THOSE DEVICE USERS AND
SOMETHING WE ALL WANT TO
DISCUSS.
SARAH AND ON —- SARAH?
>> THIS IS FANTASTIC.
IT SEEMS THAT THE THREE PEOPLE
WHO ARE HERE TALKING ABOUT THE
TRIALS IT HAS BEEN WONDERFUL
TO HEAR YOUR PERSPECTIVE.
ALL PEOPLE WHO ARE WITH YOU
DAY BY DAY BENEFIT YOU DAY BY
DAY?
NEURAL DEVICE STUDIES ARE
DIFFERENT WINSTON'S GROUP SO
IS THERE ANYTHING DIFFERENT
ABOUT WHAT IS OWED TO A
PARTICIPANT IN TERMS OF
MAINTENANCE OR UPKEEP TO
CONTINUE BENEFITS IF THEY
MAINLY USE THE DEVICE IN WAYS
THAT ARE VERY BENEFICIAL IN A
LAB SETTING?
THE DEVICE IS STILL IN
SOMEBODY SAID THAT MAYBE THE
ONLY MAKE USE OF IT IN THE LAB
SETTING BUT A LOT OF THIS
STUDIES THEY ARE THERE IT IS A
LONG-TERM INVESTMENT PEOPLE
ARE MAKING AND THEY GAIN
ABILITIES IN THAT CONTEXT IS
IT DIFFERENT ARE NOT DIFFERENT
YOUR CASES ARE SO COMPELLING
BRANDY AND TYLER SAY IT IS
LIFE-SAVING.
I DON'T SEE HOW ANYBODY CAN
NOT RESPOND TO THAT CLAIM.
BUT FOR OTHER PEOPLE THAT MAY
NOT BE SO LIFE-SAVING AS LIFE
ALTERING SO DO WE THINK THAT
TRIAL MATTERS ARE THE KIND OF
BENEFIT THEY ARE GETTING?
>> AND I YIELD BACK TO LOSS OF
FUNCTION AND THEN TO
COMMUNICATE WITH THEIR
CAREGIVER AND THEN TO APPLAUD
ONE OF THE STUDIES BECAUSE
THEY GAVE HER ANOTHER
COMMUNICATION TOOL SO THAT WAS
THINKING OF THE CONTINGENCY
PLANS SO WHAT IS THE NEXT STEP
AND THEN SUDDENLY THEY LOSE
THE ABILITY TO COMMUNICATE SO
THAT WILL BE DIFFERENT PER
TRIAL BUT LOOKING AT THOSE
TREATMENT OPTIONS ARE AND
WORKING WITH THE PARTICIPANTS
BUT THERE ARE GUIDELINES WE
CAN DO IN THE DEVELOPMENT IT
IS LIFE-THREATENING FOR THOSE
WITHOUT TREATMENT RESISTANT
DEPRESSION BUT THEN THE
ABILITY TO FEED YOURSELF IF
YOU ARE A QUADRIPLEGIC SO ALL
OF THOSE DIFFERENT FUNCTIONS
APPROACH PEOPLE IN DIFFERENT
WAYS.
>> SO WHAT IS REEMERGING IS
THE IMPORTANCE OF MAPPING AND
TO PARTICIPATE IN THIS TRIAL
AND WHAT MY LIFE AS A PATIENT
OF THIS DEVICE LOOKS LIKE THAT
SOMETHING WE HEAR CAREGIVERS
SPRING UP OR PARTNERS.
BUT THEY ALSO BRING THAT UP
THAT WOULD BE HELPFUL OF HOW
THINGS WILL LOOK LIKE BECAUSE
THEY ARE INVOLVED IN THE CARE
AND I CAN BE IN A SITUATION AT
THE MOMENT WHERE THEY WILL
PROVIDE CONSENT THINKING OF
MANY THINGS AND WITH TREATMENT
SEARCH FATIGUE AND A LONG
PROCESS THEY HAVE TRIED TO
FIND SOMETHING AND FIND
SOMETHING AND THEN YOU GET TO
THE POINT YOU ARE UNDERGOING
30 PAGE RESEARCH COORDINATOR
IT'S A DOCUMENT.
I'M ALSO A LAWYER I HAVE
LOOKED AT THIS AND I TRY TO
FIND AND THEN TO FIND OUT WHAT
WILL HAPPEN BUT AT THE END OF
THE TRIAL YOU ARE THE
INSURANCE PROVIDER BUT IT'S
EVENT HARD TO IDENTIFY AND
THAT THEY DON'T COVER IT THEN
I HAVE TO PAY FOR THAT IS A
PARTICIPANT THEN MANY TIMES
THAT IS WHAT THE DOCUMENT IS
SAYING.
THERE IS SO MUCH INFORMATION
THEY HAVE TO PROCESS AND THEN
TO ILLUSTRATE WHAT THIS WOULD
LOOK LIKE WITH A TRAJECTORY
BUT ALSO WITH CARE PARTNERS IN
THAT CONSENT PROCESS AND I
KNOW THAT THE RESEARCHERS TRY
TO DO THIS BUT MAYBE MORE
STRUCTURED WAYS TO INVOLVED IN
THE PROCESS.
>> THIS HAS BEEN A GREAT PANEL
AND A GREAT WAY TO END.
JUST A REMINDER THAT
TREMENDOUS DIVERSITY OF
DIFFERENT STUDIES THAT ARE
BEING DONE.
AND THOSE OF PATIENT
PARTICIPANTS AND TRIALS AND A
DIFFERENT SITUATIONS OF
RESEARCH TEAMS AND THAT'S THE
GREAT WAY TO GO INTO THE
DISCUSSION.
WITH THAT DIVERSITY THAT IS
SOMETHING WE WILL NEED TO KEEP
DISCUSSING I HOPE THE PANELIST
WILL STICK AROUND AND THAT
THESE VOICES HAVE BEEN SO
POWERFUL AND WE GOT SO MUCH
FROM THE DISCUSSION.
THANK YOU TO OUR PANEL.
NOW WE WILL GO TO A BREAK WE
WILL START THE SECOND PANEL AT
2:30 P.M.
WE WILL SEE YOU THERE.
WELCOME BACK, EVERYBODY.
I HOPE YOU HAD A GOOD BREAK.
IT'S MY PLEASURE TO ANNOUNCE THE
SECOND PANEL THIS AFTERNOON.
WHICH IS FOCUSING ON WHAT
DIFFERENT STAKEHOLDER GROUPS
CURRENTLY PROVIDE AND WHAT THEY
CAN OR COULD PROVIDE IN TERMS OF
THE RESEARCH-RELATED CARE NEEDS
THAT WE SO POWERFULLY HEARD
ABOUT DURING PANEL ONE.
SO WITH THAT, I WOULD LIKE TO
GIVE IT OVER TO THE
CO-MODERATORS OF THIS SECOND
PANEL, WHICH ARE DR. SHAMIR AND
DR. HSU.
>> WE'LL KICK IT OFF AND AFTER
THIS WE'LL HAVE A PANEL
PRESENTATIONS FIRST SPEAKER IS
DR. YAGNA PATHAK, ENGAGING IN
TRANSLATIONAL RESEARCH FOCUSED
ON LEVERAGING DIGITAL HEALTH
TOOLS TO IMPROVE THE CLINICAL
APPLICATION OF NEUROMODULATION
THERAPY.
PRIOR TO HER CURRENT ROLE WE
INVESTIGATED NEUROMODULAR FOR
DISEASES AND PH.D. FROM
MARQUETTE UNIVERSITY FROM
DR. CHRISTOPHER HUDSON AND
FELLOWSHIP COLUMBIA UNIVERSITY
WORKING WITH DR. SHAMIR SHA.
THANK YOU, SHERRY AND THANK YOU,
ALL.
SHERRY, I THINK YOU CAN MOVE ON
TO THE NEXT SLIDE, PLEASE.
SO I REALLY JUST WANTED TO TALK
ABOUT WE HEARD A NUMBER OF TALKS
THIS MORNING FROM PATIENTS AT
THE PATIENT ADVOCATE GROUP BUT
THE KEY STAKEHOLDERS OF THE
SITUATION INCLUDE INDUSTRY
SPONSORS, REGULATORY AND
REIMBURSEMENT BODIES AS WELL AS
CLINICAL TEAMS BUT MOST
IMPORTANTLY WE NEED TO RECOGNIZE
THAT PATIENTS ARE STILL AT THE
CENTER HERE.
THE BURDEN OF RESPONSIBILITY MAY
DEFER BASED ON THE TYPE OF STUDY
THAT WE'RE TALKING ABOUT BUT THE
EFFORT SHOULD BE TO WORK IN A
COLLABORATIVE WAY TO BEST
SUPPORT PATIENT CARE.
NEXT SLIDE, PLEASE.
SO CURRENT ITEMS FOR TREATMENT
ACCESS BEYOND THE DURATION OF
THE CLINICAL TRIAL REALLY COMES
FROM TWO SPECIFIC DOCUMENTS.
THE DECLARATION OF HELSINKI AND
THE GUIDELINES BY WHO.
THE DOCUMENTS ACCOUNT FOR
ASSESSMENTS OF WHETHER THE
TREATMENT IS EFFECTIVE AND HOW
PATIENTS WERE PREPPED FOR
PATIENTS BEYOND THE TRIAL.
AND SO IF EVERYTHING IS IDEAL,
FOR A STUDY OUTCOME, THEN THESE
ARE SOME REALLY GREAT THINGS TO
THINK ABOUT.
SO WHAT IS AN IDEAL PATIENT
CONTINUUM.
NEXT SLIDE.
IDEALLY WE EXPECT IT TO BE A
NEAR PROCESS WITH CERTAIN
PROCESSES.
THIS JOURNEY STARTS WITH THE
PATIENT PARTICIPATION IN A
CLINICAL TRIAL FOLLOWED BY OUR
REGULATORY APPROVAL AND
REIMBURSEMENT COVERAGE HOPEFULLY
LEADING TO A MARKET LAUNCH FOR
THE DEVICE OR THE SYSTEM SO THAT
THERAPY IS EVENTUALLY ACCESSIBLE
AS STANDARD OF CARE.
HOWEVER, THIS IS THE IDEAL
SCENARIO AND WE ALL KNOW THAT
SELDOM DO WE HAVE IDEAL
SCENARIOS IN REAL LIFE.
SO THE REALITY IS SOMETHING
COMPLETELY DIFFERENT.
NEXT SLIDE, PLEASE.
SO IN REALITY, THE PATIENT
JOURNEY IS NOT SO
STRAIGHTFORWARD.
WHAT REALLY HAPPENS WHEN, WHAT
HAPPENS WHEN REGULATORY APPROVAL
IS DELAYED?
THERE ARE CERTAIN OPTIONS FOR
CONTINUED PARTICIPATION IN OPEN
LABEL EXTENSION STUDIES OR EVEN
ROLLOVER CLINICAL TRIALS, IF
THIS SPONSOR WISHES TO CONDUCT
THESE ADDITIONAL EVIDENCE.
HOWEVER, THE OTHER POTENTIAL
OPTIONS THAT CURRENTLY EXIST
LIKE EXPANDED ACCESS ARE OFTEN
COUPLED WITH CONTINGENCIES
INVOLVING SEVERAL OF THE
STAKEHOLDERS THAT I MENTIONED
EARLIER.
AND SO OVERALL THERE'S REALLY A
LACK OF PROVISION FOR
INCONCLUSIVE STUDIES MAKING
THESE UNIDEAL SITUATIONS
CHALLENGING TO NAVIGATE.
WHAT HAPPENS WHEN REGULATORY
APPROVAL IS DELAYED.
WHAT HAPPENS IF A STUDY DOESN'T
REALLY MEET ITS PRIMARY
ENDPOINT?
WHAT ARE POTENTIAL OPTIONS AND
WHO REALLY BEARS THE BURDEN OF
RESPONSIBILITY HERE?
AND SO RIGHT NOW WE REALLY DO
NOT HAVE PROVISIONS TO KIND OF
ACCOUNT FOR THESE UNIDEAL
SITUATIONS.
NEXT SLIDE, PLEASE.
SO IN THIS LANDSCAPE, THERE
SHOULD BE CONSIDERATIONS FOR
CONTINUED CARE THAT REALLY COVER
CLINICAL NEEDS, TECHNOLOGY NEEDS
AND COVERAGE.
SO WE NEED TO ANSWER QUESTIONS
ABOUT HOSPITAL AND CLINICIAN
RESOURCES, SPECIFICALLY AROUND
INTEGRATING RESEARCH AND
INVESTIGATIONAL THERAPY IN
EXISTING PATHWAYS.
UNIQUE TO IMPLANTED DEVICES WE
ALSO NEED TO CONSIDER THE
CONSEQUENCES OF EXPLANTING A
DEVICE OR EXPLANTING A SYSTEM
VERSUS MAINTAINING OR REPLACING
THE SYSTEMS.
FROM A DEVELOPMENT PERSPECTIVE,
WE REALLY SHOULD BE THINKING
ABOUT HOW THESE INVESTIGATIONAL
DEVICES WILL INTERACT WITH
FUTURE SYSTEMS, WHAT HAPPENS TO
THE DATA BEYOND THE TRIAL, AND
LASTLY, WHAT IS COVERED, WHO
PAYS FOR WHICH PIECE, AND THAT
LAST PIECE IS ESPECIALLY
CHALLENGING IN LARGER CLINICAL
TRIALS THAT TEND TO BE MULTISITE
OR MULTIGEOGRAPHY BECAUSE
REIMBURSEMENT AND REGULATORY
RULES ARE OFTEN REGION SPECIFIC.
SO THESE ARE SOME BUCKET
CATEGORIES IN WHICH WE SHOULD BE
THINKING ABOUT CONTINUED CARE
AND HOW RESPONSIBILITIES SHOULD
BE DIVIDED FOR POST-TRIAL
ACCESS.
NEXT SLIDE, PLEASE.
SO REALLY THE QUESTION IS HOW
CAN WE DO BETTER?
AND I THINK THIS WORKSHOP IS A
REALLY GREAT INITIATIVE TOWARDS
SOLVING THIS PROBLEM BECAUSE WE
REALLY SHOULD BEGIN CLINICAL
TRIAL PLANNING WITH THE END IN
MIND.
SO AS OPTIMISTIC AS WE MIGHT
WANT TO BE WE SHOULD CONSIDER
ALL POTENTIAL OUTCOMES, EVEN IF
THOSE OUTCOMES ARE NOT ALWAYS
FAVORABLE OUTCOMES, BECAUSE
ULTIMATELY IT'S IN THOSE
SITUATIONS WHEN WE DO HAVE
UNFAVORABLE OUTCOMES WHERE THE
CHALLENGES ARE THE GREATEST AND
WE REALLY SHOULD BE ACCOUNTING
FOR WHAT IS BEST FOR THE PATIENT
AND I KNOW SOMEONE MENTIONED
THIS MORNING WHAT ARE INCENTIVES
FOR INDUSTRY SPONSORS, WHAT ARE
INCENTIVES FOR SOME OF THE OTHER
KEY STAKEHOLDERS INVOLVED, BUT I
DO ULTIMATELY BELIEVE THAT
EVERYONE IS IN THIS TO REALLY
IMPROVE THE OUTCOMES FOR
PATIENTS THAT WE ARE DEVELOPING
OUR TECHNOLOGIES FOR, THAT WE'RE
DEVELOPING OUR TREATMENTS FOR,
AND THAT WE ULTIMATELY WISH TO
SERVE.
SO WE REALLY SHOULD BE KIND OF
COMING BACK TO WHAT CAN WE DO TO
REALLY MAKE THIS A
PATIENT-CENTRIC APPROACH AND CAN
YOU START THINKING ABOUT SORT OF
THESE POTENTIAL OUTCOMES AT THE
BEGINNING OF THE CLINICAL TRIAL
PLANNING.
NEXT SLIDE, PLEASE.
AND SO FOR THAT WE CAN REALLY
START BY DEFINING THE PATIENT
CONTINUUM DURING PROTON
DEVELOPMENT.
WHAT DOES INFORMED CONSENT LOOK
LIKE?
HOW MANY PROCEDURES CAN WE
SUPPORT DURING THE TRIAL?
HOW MANY PROCEDURES CAN WE
SUPPORT BEYOND THE TRIAL?
WHAT ARE THE CONSEQUENCES TO THE
PATIENT IF THEY'RE ASKED TO
PARTICIPATE IN ADDITIONAL
RESEARCH?
HOW DO WE COMPLY WITH PRIVACY
AND SECURITY PRACTICES IF WE
NEED TO EXTEND DATA COLLECTION
BEYOND THE TRIAL?
SO WE HAD A PATIENT THIS MORNING
TALK ABOUT WHAT'S IN HER
CLINICAL HISTORY VERSUS REALLY
INFORMATION ABOUT HER TRIAL.
HOW DO WE BALANCE THE RESEARCH
CONTINUUM WITH CLINICAL
STANDARDS AND THAT SHOULD BE
SOMETHING THAT WE START PLANNING
FOR AND DEFINING DURING THE
PROTON DEVELOPMENT STAGE AND NOT
SORT OF AT THE END OF THE TRIAL
WHEN IT MIGHT BE TOO LATE TO ASK
FOR EXPANDED INFORMED CONSENT
FROM THESE PATIENTS OR TO
ALLOCATE MORE BUDGET.
AND SO THAT BRINGS US TO THE
NEXT POINT, WHICH IS FROM A
MINDSET PERSPECTIVE WE REALLY
NEED TO OPT FOR A PREVENTIVE OR
AN ANTICIPATORY APPROACH AND
ALIGNMENT WITH KEY STAKEHOLDERS
AND EXPLICIT DEFINITION ABOUT
POST TRIAL GROWTH AND BUDGET
BECAUSE WE ALL KNOW THAT THESE
TRIALS CAN BE VERY EXPENSIVE AND
IT'S AN INTRICATE PLAY BETWEEN A
LOT OF DIFFERENT PARTICIPANTS
THAT ARE ULTIMATELY TRYING TO
IMPROVE CARE FOR THE PATIENT.
HOWEVER, IF THERE ISN'T
SOMETHING DEFINED AHEAD OF TIME
THEN WHO SORT OF TAKES THE
BURDEN WHEN THE TIME FOR THAT
COMES TO.
SO I THINK A LOT OF THAT NEEDS
TO BE DEVELOPED FROM A
PREVENTIVE OR ANTICIPATORY
APPROACH THAT SHOULD WE HAVE
UNFAVORABLE OUTCOMES HOW DO WE
ACCOUNT FOR THAT?
AND LASTLY, WE NEED A THOROUGH
BUT ITERATIVE RISK BENEFIT
ASSESSMENT.
SO WE ALL KNOW FROM ALL OF OUR
EXPERIENCES THAT THERE ISN'T THE
ONE-SIZE-FITS-ALL FOR ALL
PARTICIPANTS.
ALL PARTICIPANTS MAY NOT BENEFIT
WITH THERAPY, AND SOME
PARTICIPANTS MAY HAVE
LIFE-CHANGING OUTCOMES AS WE
ALSO SAW THIS MORNING.
SO AS WE HAVE MORE INFORMATION
WE SHOULD REALLY STRIVE TO MAKE
DATA DRIVEN DECISIONS AND ADJUST
ACCORDING TO THE EVIDENCE THAT
WE HAVE AT HAND.
SO WE SHOULD BE DOING THIS IN A
VERY ITERATIVE WAY.
THESE SUGGESTIONS ARE ULTIMATELY
ALIGNED WITH THE EXISTING
GUIDELINES THAT I TALKED ABOUT
INITIALLY, BUT ALSO TAKE INTO
ACCOUNT NUANCES THAT ARE
PARTICULARLY ASSOCIATED WITH THE
UNIQUE CHALLENGE OF SUPPORTING
IMPLANTABLE SYSTEMS FOR TREATING
CHRONIC DEBILITATING DISORDERS
LIKE THE ONES WE DO.
WE NEED TO WORK COLLABORATIVELY
BETWEEN DIFFERENT STAKEHOLDER
GROUPS AND CONTINUE TO RECENTER
AND CONSIDER PATIENT'S BEST
INTERESTS AS WE THINK ABOUT
PROBLEM SOLVING FOR TRIALS THAT
WE ARE GOING TO BE PLANNING FOR
IN THE FUTURE.
WITH THAT, I WOULD LIKE TO END
HERE AND I CAN TAKE QUESTIONS.
THANK YOU.
I SEE USUAL HAND UP.
>> THANK YOU SO MUCH.
THAT WAS WONDERFUL.
I WAS HOPING YOU COULD SPEAK TO
A LITTLE BIT WHAT THE CURRENT
PRACTICES ARE IN MAYBE BROADLY
FROM DEVICE MANUFACTURERS,
POST-TRIAL CARE, COULD YOU SPEAK
TO THAT FOR A MOMENT.
>> THERE ISN'T ONE SPECIFIC
ANSWER TO THAT QUESTION, RIGHT.
SO I THINK WITH TRIALS THAT WE
HAVE CONDUCTED WHERE PATIENTS
NEEDED POST-TRIAL ACCESS -- AS I
SAID IT'S A COMPLEX NUANCE TOPIC
AND THERE ARE A LOT OF FACTORS
THAT GO INTO DECISION-MAKING
WHAT WE SUPPORT AND HOW WE
SUPPORT THAT.
RIGHT NOW IT'S SITUATION BASED
WHAT THE PATIENT NEEDS AND
THAT'S WHAT WE TAKE INTO ACT IN
MAKING THE DECISION HOW DO WE
SUPPORT THE PATIENT.
THAT'S WHERE IT COMES FROM, WE
KNOW FROM OUR EXPERIENCE IT'S
NOT GOING TO BE A
ONE-SIZE-FITS-ALL.
IT'S KIND OF HARD TO SAY THERE'S
A STANDARD PRACTICE BECAUSE WE
KNOW THAT A STANDARD PRACTICE
MAY NOT BE BENEFICIAL TO
EVERYONE THAT'S INVOLVED.
SO I THINK WE SHOULD COME UP
WITH A STANDARD PRACTICE AND
MAYBE THAT STANDARD PRACTICE
NEEDS TO BE DEFINED AHEAD OF
TIME SO THAT EVERYONE, SO
THERE'S ENOUGH TRANSPARENCY
BUILT IN EVEN FOR THE PATIENTS
THAT ARE PARTICIPATING IN THESE
TRIALS SO THEY KNOW KIND OF WHAT
TO EXPECT IF THINGS DON'T GO AS
WE ALL HOPED THEY WOULD GO.
BECAUSE I THINK WE ALL TEND TO
BE VERY OPTIMISTIC BY NATURE
WHICH IS GOOD.
BUT YOU SHOULD ALSO KIND OF PLAN
FOR THE REALISTIC SCENARIOS.
>> THAT'S GREAT.
MAYBE WE'LL KEEP MOVING.
THERE'S GOING TO BE I THINK A
BUNCH OF QUESTIONS THAT CAN
LIGHT -- FURTHER FLESH OUT HOW
THAT PARTICIPATION MIGHT LOOK
LIKE.
I THINK IT WOULD BE GREAT TO
COME BACK TO THAT.
>> THAT'S GREAT.
THANKS AMIR.
>> LET ME GO AHEAD AND
INTRODUCE OUR SECOND SPEAKER OF
THE SESSION.
PLEASURE TO INTRODUCE DR. MARTY
MORRELL FROM STANFORD
UNIVERSITY.
HAS ACTIVELY BEEN INVOLVED IN
HELPING TO BRING NEW MEDICAL AND
DEVICE THERAPIES TO PATIENTS
WITH EPILEPSY.
SINCE 2004, SHE SERVED AS THE
CHIEF MEDICAL OFFICER AT
NEUROCASE A COMPANY THAT
DEVELOPED A RESPONSIVE
NEUROSTIMULATE TER FOR TREATMENT
OF UNCONTROLLABLE PARTIAL
SEIZURES.
PAST PRESIDENT AND PRINCIPAL
INVESTIGATOR OF A GRANT FOR
RESPONSE FOR LINUX GASTO
SYNDROME, RARE FORM OF EPILEPSY.
THANK YOU.
>> THANK YOU SO MUCH, SAMIR.
AND I'M SO PLEASED BECAUSE YOU
NEVER WANT TO COME AND SPEAK
WHEN YOU'RE SAYING THE SAME
THING AS EVERYBODY ELSE, BUT I
THINK THERE'S GOING TO BE A FAIR
AMOUNT OF THAT, WHICH I BELIEVE
IS GOOD IN THIS CONTEXT.
SO JUST TO TELL YOU, I HAVE SOME
POTENTIAL CONFLICTS WHICH I'LL
MAKE VERY APPARENT.
I'M CHIEF MEDICAL OFFICER NEURO,
AS SAMIR SAID AND I'M ALSO AT
STANFORD AND I'M BEING FUNDED
FORTUNATELY BY NIH.
THESE ARE MY OWN VIEWS.
THEY DON'T REPRESENT ANYBODY
ELSE'S.
BUT LET ME GIVE YOU MY
PERSPECTIVE.
I AM FIRST AND FOREMOST A
PHYSICIAN.
I'M A NEUROLOGIST WHO IS SPECIAL
TRAINED IN EPILEPSY.
AND SPEND THE MAJORITY OF MY
CAREER IN ACADEMICS AS A
CLINICIAN TEACHER AND
TRANSLATIONAL RESEARCH PERSON.
I THEN, AS PART OF ALL OF THAT
TRANSITIONED INTO INDUSTRY WITH
A STARTUP DEVELOPING A NEW
PRODUCT FOR THE DISORDER THAT I
WAS TREATING IN MY PATIENTS
EPILEPSY.
I CONSIDER MYSELF A PATIENT
ADVOCATE.
I'VE BEEN VERY INVOLVED WITH
PATIENT ADVOCACY ORGANIZATIONS
AND I AM ALSO NOW IN THE SECOND
PART OF MY CAREER VERY SENSITIVE
TO WHAT IT TAKES TO BRING
SOMETHING ENTIRELY NEW TO
PATIENTS AND HOW HARD THAT IS.
SO I MAY ACTUALLY EMPHASIZE A
LITTLE BIT MORE THE SMALL
COMPANY PERSPECTIVE WHICH MAY
NOT BE AS FAMILIAR WITH
EVERYONE.
BUT WHAT DO WE HAVE HERE?
I WAS READING THE PREWORK THAT
WAS DONE, A LOT OF STUFF ABOUT
SHARED BURDEN, BUT IT'S ALSO A
SHARED OPPORTUNITY.
AND THE OPPORTUNITY FOR THE
PATIENT AND HER LOVED ONES IS
THAT THE TREATMENT WILL BRING
HEALTH AND BETTER QUALITY OF
LIFE.
AND FOR THE ACADEMIC RESEARCH
INSTITUTE, THAT'S A BUSINESS, AS
MUCH AS ANYTHING ELSE, BUT OF
COURSE IT'S MISSION DRIVEN
BUSINESS.
FOR PHYSICIANS, ALTHOUGH
OBVIOUSLY PHYSICIANS MAKE MONEY,
THEY HAVE TO, I THINK MOST
PEOPLE GO INTO HEALING
PROFESSIONS BECAUSE THEY'RE
MOTIVATED TO HELP REDUCE
SUFFERING AND SOCIETY RECOGNIZES
THAT IT'S NOT JUST THE MONEY,
IT'S THE COST OF MISSED
OPPORTUNITIES.
IT'S THE LIFE THAT IS NOT LIVED.
AND FOR INDUSTRY, IN ORDER TO
SURVIVE MUST BE A COMMERCIALLY
VIABLE PRODUCT BUT I WOULD SAY
PEOPLE CAN DO VIDEO GAME
SOFTWARE OR HEALTHCARE
TECHNOLOGY THERE'S THAT
MOTIVATION.
FOR INSURERS, I THINK THE
MOTIVATION IS REALLY TO MAINTAIN
THE HEALTH OF THIS SUBSCRIBER
AND THAT OF COURSE REDUCES
MEDICAL COSTS.
THERE ARE FINANCIAL INCENTIVES
AND ALSO A MISSION AND ALTRUISM
AND I THINK THE MOTIVATION TO
BENEFIT INDIVIDUALS IN SOCIETY
IS SOMETHING THAT IS UNIFYING.
WE HAVE A SHARED RESPONSIBILITY.
NOW I WANT TO SHARE ABOUT THE
COMPANY I'M WORKING WITH TO SET
IS CONTEXT.
THIS IS A COMPANY THAT WAS
FOUNDED IN 1997 THE CONCEPT AND
PROVIDE THEM TO AN HE SOUGHTIC
DISORDER EPILEPSY AND THE
COMPANY SPENT A YEAR DOING
DEVELOPMENT AND PROOF OF CONCEPT
SO BY 2002 THERE WAS AN
IMPLANTABLE DEVICE THAT COULD BE
TESTED IN 2004 THE TRIALS BEGAN
OF FIRST ENDED UP 256 PEOPLE
IMPLANTED.
THESE INDIVIDUALS WERE FOLLOWED
FOR NINE YEARS.
AND IN 2010 AN APPLICATION WAS
APPLIED FOR AN APPROVAL
PREMARKET APPROVAL APPLICATION
FOR FDA'S CLASS 3 IMPLANTABLE
DEVICE.
FOR HUMOR, 2010 FDA WANTED
EVERYTHING IN PAPER COPIES AND
THREE COPIES.
SO THIS IS WHAT IT LOOKED LIKE,
AND THAT'S THAT.
AND OF COURSE IT BEING A NOVEL
DEVICE, THERE WAS AN ADVISORY
PANEL IN 2011 AND THEN DOING
THIS THE RIGHT WAY THEN FINALLY
IN 2003, NOVEMBER, THE END OF
NOVEMBER, FDA PROVIDED APPROVAL
FOR PARTIAL SEIZURES.
AND THEN IN 2015, WE WERE ABLE
TO HAVE OUR TWO-YEAR PATIENT
CELEBRATION WHICH WAS VERY
MOVING.
NOW, WHAT DOES THIS MEAN WITH
16 YEARS?
AND OVER $350 MILLION IN A
COMPANY THAT EXISTED ONLY FOR
THIS.
AND SO THERE WERE MANY NEAR
DEATHS ALONG THE WAY FOR A
NUMBER OF REASONS, EVEN THOUGH
THE RESULTS WERE VERY STRONG.
THERE WAS JUST A LONG TIME, A
LOT OF MONEY, A LOT OF RISK.
MY OTHER STORY IS TO TALK ABOUT
THE APPLICATION OF THE
TECHNOLOGY TO A TERRIBLE
DISORDER.
AND THIS IS AN INVESTMENT THAT
NIH IS MAKING.
SUPPLYING APPLYING THIS
TECHNOLOGY AND APPLYING IT TO A
EPILEPSY CALLED LINUX GASTO,
INJURY AND HIGH RISK OF DEATH
AND THERE AREN'T ANY OTHER
OPTIONS.
THIS IS NOT ONLY TERRIFICALLY
IMPACTFUL TO PATIENT BUT ALSO
CAREGIVERS.
SO OTHER SOLUTIONS ARE NEEDED.
THIS IS A RARE DISEASE.
IT'S AN ORPHAN INDICATION.
THERE IS NO COMMERCIAL
MOTIVATION TO DELIVER A THERAPY
FOR THIS PATIENT POPULATION, BUT
THERE SURE IS THE CLINICAL
MOTIVATION, THE ALTRUISM, THE
WANT TO HELP EASE SUFFERING.
SO A GRANT WAS SUBMITTED WITH
SIX ACADEMIC MEDICAL CENTERS TO
APPLY SOME OF THE DEVICE OF THIS
DEVICE, AND IT WAS TERRIFIC.
IN MARCH 2020, NIH INDICATED
THAT FUNDING WAS LIKELY BUT DID
SAY THAT PROBABLY IT WOULD
REQUIRE AN ACCEPTABLE PLAN TO
PROVIDE POST STUDY CARE.
AND SO OUR PROJECT OFFICERS,
FABULOUS, SAID, SEE WHAT YOU CAN
DO.
SO WE EXPLORED OPTIONS.
WE COULD NOT GUARANTEE THAT
AFTER THIS TRIAL WE WOULD BE
FINANCIALLY ABLE TO PROVIDE CARE
BECAUSE EVEN AT THAT POINT YOU
DON'T EVEN KNOW IF COMPANY IS
GOING TO BE VIABLE.
NEUROPATIENTS IS OKAY, BUT FOR
YOUNG COMPANIES, WHAT IF IT
DOESN'T EXIST ANY LONGER?
AND IN GOING TO EACH OF THE
ACADEMIC INSTITUTIONS AND
HOSPITALS, THEY WERE NOT WILLING
TO COMMIT TO ANY FUNDS IN THE
FUTURE AND WE REACH OUT TO
ENSURER, EVEN OUTSIDE THE UNITED
STATES, THE ONES THAT INSURE
JENNIFER ANISTON'S HAIR AND
EARTHQUAKES, THESE RARE EVENTS,
TO SEE IF THEY WOULD BE WILLING
TO CREATE A POLICY FOR POST
STUDY CARE THAT NEUROPACE COULD
PAY FOR.
BUT THERE'S NO PRECEDENT FOR
THAT.
AND IT WAS NOT POSSIBLE TO GET A
SUPPLEMENT TO THE NIH GRANT.
NIH TO PROVIDE FOR THIS AND WE
COULD GO TO CMS BUT THEY HAVE NO
MECHANISM FOR ESTABLISHING POST
STUDY CARE EITHER.
SO I FELT LIKE THERE WAS ALL OF
THIS GOING ON.
AND WE ALL WANTED THE SAME
THING.
SO THAT'S NOT GOING TO WORK.
SO WE HAVE TO FIGURE OUT HOW TO
DO THIS.
AND WHAT HAPPENED?
WELL, IT WAS THE YEAR OF GETTING
TO SOMEPLACE OF AGREEMENT.
AND EVERYONE WAS SATISFIED THAT
THESE INDIVIDUALS HAVE TERRIBLE
EPILEPSY, THEY WILL BE GETTING
ROUTINE EPILEPSY CARE SO REGULAR
ACCESS TO PHYSICIANS WILL BE
COVERED.
NEUROPACE COMMITTED TO DONATING
THE DEVICE AND PROVIDING SUPPORT
FOR THE DEVICE.
IT'S NOT COVERED BY THE INSURER.
AND THE PARTICIPATING PHYSICIANS
SAID THEY WOULD PROVIDE
PROGRAMMING FOR THE CHARGE FOR
UNINSURED PATIENTS.
HOWEVER, THE INSTITUTIONS AND
THE HOSPITALS WERE NOT WILLING
TO DO THAT.
BUT THOUGH DID SAY AND PUT IN
WRITING THEY WOULD AGREE TO WORK
VERY HARD TO GET THIS.
AND THEN FINALLY THE PATIENT AND
IN THIS CASE MOSTLY THEIR FAMILY
MEMBERS DO HAVE IN THE CONSENT
AND ASCENT, THAT IF ALL THE
INSURANCE OPTIONS ARE EXHAUSTED
THEN THERE MAY BE INDIVIDUAL
RESPONSIBILITY FOR COSTS.
SO IN THIS CASE MOST EVERYBODY
HAS A PIECE OF THE SOLUTION.
BUT IT TOOK A LONG TIME.
SO IT WAS WELL OVER A YEAR FROM
THE TIME THAT WE HEARD THAT IT
WAS LIKELY THAT THE GRANT WOULD
BE FUNDED UNTIL WE RECEIVED OUR
YOU
YOUNGEST SINGLE AWARD.
IT WAS NOT FOR THE LACK OF THE
NIH TEAM HELPING AND TRYING THEY
ABSOLUTELY DID.
BUT IN THE MEANTIME THESE ARE
PEOPLE WITH NO TREATMENT WHO
WERE DYING FROM THEIR DISEASE.
SO IT'S HAVING THINGS TOGETHER
AHEAD OF TIME IN SOME
UNDERSTANDING.
TIME IS REALLY OF THE ESSENCE.
SO WHO SHOULD PAY FOR POST STUDY
CARE IF THE DEVICE IS NOT
APPROVED?
OR THE COMPANY DOESN'T EXIST.
WE JUST HEARD A WONDERFUL
PRESENTATION.
THAT'S ABBOTT.
THEY'RE NOT GOING TO SEIZE TO
EXIST BUT A COMPANY LIKE
NEUROPACE CERTAINLY COULD.
SO IF IT'S A PROFITABLE COMPANY
THEN YOU CAN EXPECT THE COMPANY
TO OFFSET I BELIEVE FROM SOME
EXTENT TO OTHER REVENUES.
IF IT'S NONPROFIT TABLE
NONEXTENT COMPANY, THEN THE ONLY
WAY THE COMPANY CAN DO
SOMETHING, PUT IT ASIDE BOOK FOR
THE TRIAL.
AND SO I BELIEVE THAT IT IS
ACTUALLY REASONABLE WHEN SMALL
COMPANIES OR BIG COMPANIES ARE
DOING STUDIES TO PLAN AHEAD OF
TIME FOR MAKING SOME KIND OF
COMMITMENT FOR THE THINGS DON'T
GO WELL.
FOR MEDICAL INSURERS, IF THE
PATIENT BENEFITS, THEN I THINK
IT'S IN MY OPINION.
TO CONTINUE ON THE CARE.
IF THE PATIENT DOESN'T BENEFIT,
I WOULD HOPE THAT THE MEDICAL
CARE INSURER WOULD PARTICIPATE
IN COVERAGE FOR THE DEVICE
EXPLANT.
AND ACADEMIC INSTITUTIONS, THEY
BENEFIT FROM THE TRIAL.
THE ACADEMIC INSTITUTIONS ARE
OUR BIG INSTITUTIONS.
THEY ARE OUR ENTERPRISES.
THEY'RE COMPANIES.
THEY MAKE MONEY.
THAT THEY THEN PUT BACK INTO
YOUR MISSION.
BUT IF THEY ARE GOING TO MAKE
THAT COMMITMENT, THEN I BELIEVE
THERE'S SOME SHARED
RESPONSIBILITY FOR OUTCOMES.
AND THAT WOULD BE PERHAPS
PROVIDING PARTIAL CHARGES FOR
HOSPITAL PHYSICIAN SERVICES AND
THEN FOR PATIENTS I BELIEVE AS
WE HAVE IN OUR CONSENT FORM,
THERE'S BEEN A LOT OF DISCUSSION
ABOUT THAT THERE IS A PATIENT
AND FAMILY MEMBER PIECE OF THIS
AS HARD AS THAT IS SOMETIMES TO
THINK.
BUT CERTAINLY PEOPLE DO NEED TO
UNDERSTAND WHAT IS THE POTENTIAL
OUTCOME OF A NONAPPROVED DEVICE.
SO AND THEN GOVERNMENT FUNDERS.
AND WE'VE TALKED ABOUT NIH, BUT
FDA TOO.
FDA'S ORGANIZED TO PROTECT
PATIENT, PUBLIC SAFETY.
AND THEY DO ABSOLUTELY A
WONDERFUL JOB.
BUT THEY ALSO ARE DEVOTED TO THE
HEALTH OF THE PUBLIC.
AND PROBABLY RESEARCH IS PART OF
THAT.
AS IT IS FOR THE DEPARTMENT OF
DEFENSE, AS IT IS FOR MEDICARE
AND OTHERS.
WHAT WE NEED ARE CERTAINLY
ETHICAL PRINCIPLES GUIDING THIS,
AND THOSE HAVE GOT TO BE CORE.
BUT WE NEED PRACTICING PLAT MAT
TICK SOLUTIONS, I BELIEVE
EVERYONE IS INVOLVED IN THIS AND
ENJOYS THE GLORY.
THANKS, I'LL TURN IT OVER TO
MARTY
>> THANKS FOR THE OVERVIEW OF
THE SUBJECT.
WE HAVE TIME FOR ONE QUICK
QUESTION IF ANYBODY HAS ONE.
>> SARA.
>> INTERESTING, ALL THE FINGERS
ARE POINTING, THEY WANT EVERYONE
TO PAY.
AND YOU LIST THAT NEUROPACE
COVERS THE DEVICE IF IT'S NOT
COVERED BY INSURANCE.
AND THE UNIVERSITY HOSPITAL
DOING SOMETHING MAYBE NOT QUITE
DOING IT.
I'M WONDERING BECAUSE I DON'T
KNOW THE FIGURES, WHAT'S THE
PROPORTIONAL COST OF WHAT IT IS
TO PROVIDE THIS CARE, SO HOW
MUCH OF IT IS COVERED BY
NEUROPACE, HOW MUCH ARE THE
DEVICES RELATIVE TO THE CARE
THAT MIGHT BE NEEDED FOR YEARS
AND YEARS?
YOU KNOW THE COST OF SOMETHING
IS VERY HARD TO DETERMINE
BECAUSE THERE ARE THE COSTS OF
MANUFACTURING THE DEVICE.
BUT MOST OF THE COSTS ARE
RELATED TO THE PROCEDURE TO
IMPLANT THE DEVICE AND WHICH IS
THE SURGICAL PROCEDURE AND THE
BRIEF HOSPITALIZATION AND THEN
THE MANAGEMENT OF THE DEVICE,
THE MEDICAL CARE.
SO THERE ARE -- AND THE COSTS OF
THE SURGERY AND THE OTHER
PROCEDURES ARE DETERMINED BY THE
HOSPITAL AND THE ACADEMIC
INSTITUTION.
THEY SET THOSE COSTS.
SO A COMPANY LIKE NEUROPACE OR
ABBOTT OR MEDTRONIC OR THE
TINIEST COMPANY HAS NO CONTROL
OVER WHAT THE MEDICAL COSTS
ACTUALLY ARE BECAUSE THOSE ARE
NOT SET.
SO THEY HAVE CONTROL OVER THE
DEVICE AND THEY HAVE CONTROL
OVER WHAT EVER SUPPORT IF
THEY'RE CLINICAL ENGINEERS AND
PEOPLE IN OR WITH THE DEVICE
THEY HAVE CONTROL OVER THAT.
BUT THEY HAVE NO CONTROL OF ANY
OF THE OTHER, WHICH IS WHY IT
HAS TO BE DONE MULTIPLE PARTIES.
>> AND THE ANALYSIS GROUP IN
THE CENTER FOR CLINICAL
STANDARDS AND QUALITY.
PART OF THE CENTERS FOR MEDICARE
AND MEDICAID SERVICES ON THE
MEDICARE SIDE.
DR. LI.
>> GOOD AFTERNOON.
WANT TO THANK YOU FOR THE
OPPORTUNITY TO TALK ABOUT THE
MEDICARE PERSPECTIVE ON
POST-TRIAL CARE.
I SEE THE SLIDES ARE COMING UP.
I'LL GO AHEAD AND START.
TALKING.
AND NEXT SLIDE.
I WANTED TO GET THIS PARTICULAR
DISCLAIMER THIS PRESENTATION IS
NOT A LEGAL DOCUMENT.
IF THERE'S FURTHER QUESTIONS
ABOUT WHAT MEDICARE PROVIDERS,
THE CRITERIA ARE, SO IT'S
COVERAGE DECISIONS, THEN ONE
WOULD HAVE TO GO BACK LOOK AT
THE RULES AND REGULATIONS
RELATED TO THAT QUESTION.
SO NEXT SLIDE, PLEASE.
SO OUR WORK AT MEDICARE, I WORK
AS PREVIOUSLY DESCRIBED COVERAGE
ANALYSIS GROUP.
WHAT THAT MEANS IS THAT MY GROUP
DETERMINES WHAT ITEMS OR
SERVICES SHOULD BE COVERED AND
THEREFORE REIMBURSED AND THEN
WHAT I'M GOING TO DO IS DESCRIBE
THAT PROCESS OF COMING TO GET A
DECISION.
AND SO UNDERNEATH ALL OF THIS IS
THE CONTRACT THAT OUR AGENCY WAS
ESTABLISHED BY THE SOCIAL
SECURITY ACT OF 1965.
AND HOW WE DETERMINE WHAT'S
COVERED OR WHAT'S REIMBURSED IS
BASED ON THE EVIDENCE AND THE
EVIDENCE SHOWS THAT THE BIAS,
THE ITEM OR DEVICE IS REASONABLY
NECESSARY FOR DIAGNOSIS
CONFIDENT TREATMENT OR INJURY OR
TIM PROVES THE FUNCTION OF A
MALFORMED BODY.
AND NEXT COUPLE OF SLIDES I'LL
DESCRIBE HOW TO DO THAT.
NEXT SLIDE, PLEASE.
AND SO FIRST QUESTION IS WHAT IS
ACTUAL COVERAGE DETERMINATION.
THIS IS HOW WE DETERMINE WHAT
SHOULD BE COVERED, WHICH ITEM OR
DEVICE SHOULD BE COVERED ON A
NATIONAL BASIS.
SO WHAT WE DO BRIEFLY IS REVIEW
THE PUBLISHED EVIDENCE TO
DETERMINE WHETHER OR NOT THAT
ITEM OR DEVICE IN FACT IMPROVES
A HEALTH OUTCOME THAT'S
MEANINGFUL TO THE MEDICARE
POPULATION.
SO WE GO AHEAD REVIEW THE
PUBLISHED EVIDENCE.
LOOK FOR QUALITY, STRENGTHEN THE
TOTAL OF THE EVIDENCE, WHETHER
TO DETERMINE WHAT QUALITY IT'S
AT, WHAT TYPE OF STRENGTH IT HAS
AND WHETHER OR NOT IT IMPROVES
MEANINGFUL PATIENT-CENTERED
OUTCOMES.
AND TYPICALLY OUR GOLD STANDARD
OUTCOME IS MORTALITY THOUGH
DEPENDING ON THE ITEM OR DEVICE
WE'LL USE OTHER OUTCOMES.
NEXT SLIDE, PLEASE.
AND AS PART THAT HAD NATIONAL
COVERAGE DETERMINATION WE WILL
GO THROUGH THE EVIDENCE IF WE
FIND THE PUBLISHED EVIDENCE IS
INSUFFICIENT TO MAKE A
APPROPRIATION DECISION THE
DEVICE TO COVER OR NOT COVER IT.
WE HAVE A SEPARATE PATHWAY WHERE
WE WILL SUPPORT A CLINICAL TRIAL
WHICH THE CLINICAL TRIALS
DEVELOPING THE EVIDENCE TO HELP
US BETTER UNDERSTAND WHETHER OR
NOT THE ITEM WAS SERVICE RELATED
TO THE OUTCOME OF THE MEDICARE
POPULATION.
THOSE CLINICAL TRIALS COULD
INCLUDE RANDOMIZED OBSERVATIONAL
STUDIES REGISTRATION STUDIES AND
JUST THOSE DIFFERENT TYPES OF
CLINICAL TRIALS.
AND SO NEXT SLIDE PLEASE.
THE EXAMPLE I WANT TO USE TODAY
IS THIS PARTICULAR NATIONAL
COVERAGE DETERMINATION WHICH WE
HAD COMPLETED A NUMBER OF YEARS
AGO IT'S LOOKING AT VAGUS NERVE
STIMULATION OR TRD.
AND SO NEXT SLIDE, PLEASE WE
LOOK AT EVIDENCE AND DETERMINE
THAT LOOKING AT OVERALL QUALITY
OF THE EVIDENCE THAT THE
EVIDENCE IS REALLY PROMISING BUT
NOT CONVINCING.
CONCERNS ABOUT THE QUALITY IN
TERMS OF EPIDEMIOLOGIC ISSUES,
FOUND THE STRENGTH OF EVIDENCE
WAS NOT STRONG ENOUGH FOR US TO
ALLOW US TO MAKE AN APPROPRIATE
DECISION WHETHER OR NOT VNS OR
VAGUS NERVE THAT TREATS
TREATMENT DEPRESSION AS A RESULT
WE PROPOSE THE PATHWAY COVERAGE
WITH EVIDENCE DEVELOPMENT.
NEXT SLIDE, PLEASE.
AND SO WHAT THAT MEANS IS THAT
THE NEXT COUPLE OF SLIDES IS THE
POLICY THAT I WILL NOT GO
THROUGH THE EVIDENCE THAT WAS A
LONG PROCESS TO GET TO THIS
POINT.
BUT SO IN ESSENCE WE DECIDED WE
WOULD COVER FDA APPROVED VAGUS
NERVE STIMULATION DEVICES FROM
TREATMENT RESISTANT DEPRESSION
THROUGH THIS PATHWAY WE CALLED
COVERAGE WITH EVIDENCE
DEVELOPMENT.
AND SO WHEN DNS IS OFFERED FROM
A CNS APPROVED CLINICAL TRIAL AT
LEAST ONE YEAR WE COULD COVER
THE DEVICE AND SERVICES DURING
THE DURATION OF THE RANDOMIZED
CLINICAL TRIAL.
HOWEVER, WE WOULD ALSO -- WHAT
WE DID WAS MAKE SURE THE
CLINICAL TRIAL WAS VALID AND
WELL-DESIGNED ONE IN THE SENSE
WE HAD ONLY COVERED THE STUDY IF
IT, IF THE MAIN RESEARCH
QUESTION, THE PURPOSE OF THE
STUDY WAS TO ADDRESS WHETHER OR
NOT DNS IMPROVES HEALTH OUTCOMES
FOR TRD PATIENTS.
SO WE REQUIRED THAT THE CLINICAL
STUDIES ANSWER THERE'S A LARGE
NUMBER OF RESEARCH QUESTIONS.
I ONLY PUT TWO OF THEM HERE.
10 OR 15 QUESTIONS.
IN TERMS OF BREVITY.
THE MAIN QUESTION IS THAT WE
WANT THE CLINICAL TRIAL TO
ANSWER IS WHAT WAS THE RATE OF
RESPONSE OF USING THE VNS DEVICE
TO TREAT TREATMENT RESISTANT
DEPRESSION PATIENTS.
ANOTHER MAIN QUESTION WHAT IS
THE RATE OF REMISSION WHEN THE
VNS DEVICE IS USED FOR TREATMENT
RESISTANT DEPRESSION.
SO THERE'S A WHOLE LIKE I SAID
THERE'S A WHOLE LARGER NUMBER OF
OTHER QUESTIONS THAT WERE VERY
IMPORTANT BUT THE POINT WAS THE
PURPOSE OF THIS STUDY WAS THAT
IT HAD TO ANSWER THE QUESTION OF
WHETHER OR NOT DNS IN FACT
IMPROVES HEALTH OUTCOME OF
MAJORITY OF PATIENTS.
NEXT SLIDE, PLEASE.
AS PART OF THE DECISION, WE ALSO
INCLUDE PATIENT CRITERIA THAT IS
THE PATIENT HAD TO REACH CERTAIN
ENROLLMENT CRITERIA TO BE A PART
OF THE CLINICAL TRIAL.
SO THE PATIENT HAD TO BE, HAD TO
HAVE HAD A MAJOR DEPRESSIVE
EPISODE FOR AT LEAST TWO YEARS
OR MORE IN THE PAST TWO AND A
HALF YEARS, TWO YEARS.
THE PATIENT WOULD ALSO HAVE TO
HAVE HAD FOUR PRIOR FAILED
TREATMENTS OF ADEQUATE DOSE AND
DURATION OF THE DRUG TO QUALIFY
FOR THIS PARTICULAR STUDY.
AND JUST SKIPPING DOWN TO THE
LAST BULLET THAT I THINK THIS
PARTICULAR CRITERIA IS RELEVANT
TO THE WORKSHOP IN TERMS OF
POST-TRIAL RESPONSIBILITIES.
AT LEAST FROM THE MEDICARE, FROM
MY AGENCY'S PERSPECTIVE.
SO INDIVIDUALS WHO RECEIVE THE
PLACEBO VNS WILL BE OFFERED
ACTIVE VNS AT THE END OF THE
TRIAL.
AND SO WHAT THAT MEANS IS THAT
AFTER THE CLINICAL TRIAL IS
DONE, THOSE INDIVIDUALS IN THE
CONTROL GROUP, THOSE WHO WERE
GETTING A PLACEBO VNS DEVICE
WILL BE OFFERED THE ACTIVE VNS
DEVICE, THEY WOULD RECEIVE THE
DEVICE THAT WAS BEING USED.
THIS WOULD BE AT THE END OF THE
TRIAL.
AND THEN THIS WAS WRITTEN INTO
THE DECISION, THE DETERMINATION.
NEXT SLIDE, PLEASE.
AND SO AS I STATED BEFORE, PART
OF THIS DECISION WE INCLUDE
WHAT'S CALLED A CD PATHWAY,
COVERAGE DEPENDENT PATHWAY THE
STUDIES HAD TO MEET 13 CRITERIA.
BELOW THERE WERE ONLY FOUR OF
THOSE CRITERIA AND I INCLUDED
TWO OF THE CRITERIA THAT WE
CONSIDERED.
NOT JUST CONSIDERING OF IMPORT
BUT WE CONSIDERED IT KEY TO THE
CLINICAL TRIAL.
SO AS I STATED BEFORE, THE FIRST
BULLET, THE PRINCIPAL PURPOSE OF
THE STUDY IS TO TEST WHETHER OR
NOT THE ITEM OR SERVICE
MEANINGFULLY IMPROVES THE HEALTH
OUTCOMES OF AFFECTED
BENEFICIARIES WHO ARE
REPRESENTED BY THE SUBJECT.
SO CLEARLY THE STUDY HAD TO SHOW
IMPROVEMENT IN HEALTH OUTCOME.
IN THIS CASE IT WAS THE RATE OF
RESPONSE OF THE RATE OF
REMISSION WITH USE OF THE VNS
DEVICE VAGUS NERVE STIMULATION
DEVICE IN TERMS OF ADDRESSING
TREATMENT RESISTANT DEPRESSION.
AND IF YOU LOOK AT THE FOURTH
BULLET, STUDY DESIGN IS
METHODOLOGICALLY APPROPRIATE IN
ANTICIPATED NUMBER OF THE
SUBJECTS SUFFICIENT TO ANSWER
THE RESEARCH QUESTIONS BEING
ASKED ACTUAL COVERAGE
DETERMINATION.
WHAT THAT MEANS IS THAT WE WOULD
REVIEW THESE CLINICAL TRIALS,
WHETHER IT'S A REGISTRY OR
DOUBLE BLIND RANDOMIZED CLINICAL
TRIAL TO MAKE SURE THE
METHODOLOGY WAS APPROPRIATE.
THAT IS, THAT THERE'S COMPARISON
GROUP.
THERE'S A CONTROL GROUP SO THAT
YOU CAN COMPARE THE EFFECT OF
THE VNS DEVICE IN TREATMENT
RESISTANT DEPRESSION PATIENTS
AND THERE'S DOUBLE BLINDING IF
IT'S RANDOMIZED CLINICAL TRIAL.
IF THE REGISTER THAT IT HAD A
LARGE ENOUGH CATCHMENT AREA TO
GET A SENSE OF WHAT'S HAPPENING
NATIONALLY.
WE'LL TALK ABOUT SAMPLE SIZE.
WE WANT TO MAKE SURE THAT THE
STUDY WAS POWERED ADEQUATELY TO
ANSWER THE VARIOUS RESEARCH
QUESTIONS, A SAMPLE SIZE WAS
LARGE ENOUGH, ENROLLMENT WAS
LONG ENOUGH.
FOLLOW-UP WAS APPROPRIATE TO
MAKE SURE THAT THERE'S ADEQUATE
FOLLOW-UP FOR AS MANY PATIENTS
AS POSSIBLE AND WE LOOKED AT A
LOT OF THE OTHER VARIOUS
HEMATOLOGIC QUESTIONS CONCERNS
ABOUT THE LIMITATIONS THAT MIGHT
BE EXISTING IN THESE CLINICAL
TRIALS.
NEXT SLIDE, PLEASE.
AND THESE LAST TWO OR LAST THREE
BULLETS, I THINK, ALSO ADDRESS,
I THINK, THE QUESTIONS RAISED IN
THIS PARTICULAR WORKSHOP RELATED
TO POST-TRIAL RESPONSIBILITIES.
AT LEAST FROM THE CMS
PERSPECTIVE.
IF YOU LOOKED AT THE FIRST
BULLET.
I'LL GO AHEAD AND READ IT.
WE'RE THINKING OF NONCOVERAGE
EDUCATION.
THE FIRST TWO BULLETS.
THE FIRST ONE IS DNS IS
NONCOVERED FOR THE TREATMENT OF
TRD WHEN FURNISHED OUTSIDE THE
CMS STUDY.
MEANING THAT IF VNS IS BEING
USED AT A STUDY WE HAD NOT
LOOKED AT HAD NOT APPROVED
YET -- THANK YOU -- THIS LAST
SLIDE, THEN IT'S OUTSIDE OF THE
CN APPROVED CD STUDY WE'RE NOT
GOING TO COMPLEMENT IT.
THAT MAKES SENSE HADN'T LOOKED
AT THE CD STUDY THE CLINICAL
TRIAL THAT WE WILL NOT COVER THE
DEVICE.
THE SECOND BULLET IS INDICATIONS
OF VNS FOR TREATMENT OF
DEPRESSION NOT NATURALLY COVERED
THAT IS IF THE DEVICE IS BEING
USED OUTSIDE OF TREATMENT OF
RESISTANT -- OUTSIDE OF
TREATMENT RESISTANT DEPRESSION,
THAT WE'RE NOT GOING TO COVER
THE DEVICE.
THE LAST BULLET IS PATIENTS ARE
IMPLANTED WITH A VNS DEVICE FOR
GRD MAY RECEIVE A VNS DEVICE
REPLACEMENT IF IT'S REQUIRED DUE
TO END OF BATTERY LIFE OR ANY
OTHER DEVICE-RELATED MALFUN
MALFUNCTION, MEANING -- I THINK
THIS IS RELEVANT FOR THIS
PARTICULAR WORKSHOP -- IF THE
VNS DEVICE NEEDS TO BE REPLACED
BECAUSE THE BATTERY PROBLEMS OR
ANOTHER DEVICE RELATED
MALFUNCTION, CMS WILL REPLACE
THE DEVICE.
NEXT SLIDE, PLEASE.
SO I THINK WE SHOULD BE ON THE
LAST SLIDE.
NEXT SLIDE.
ANY QUESTIONS?
>> THERE'S TIME FOR ONE
QUESTION.
>> OKAY.
>> CAN I ASK IT?
>> GO AHEAD.
>> SO I JUST WANTED TO MAKE
SURE -- THANK YOU DR. LI FOR
THAT PRESENTATION.
MAKE SURE I UNDERSTAND.
IF A DEVICE IS IN A TRIAL THAT
COMES UNDER CED, THEN MEDICARE
WILL PAY FOR IT.
IF IT'S ALREADY, IF THE DEVICE
HAS ALREADY BEEN APPROVED UNDER
A NATIONAL COVERAGE
DETERMINATION THEN CMS OR
MEDICARE WILL PAY FOR IT.
WHAT ABOUT THE KINDS OF
SITUATIONS THAT PEOPLE ARE
TALKING ABOUT EARLIER WHERE IT'S
IN A TRIAL THAT'S OVER, THAT IT
IS NOT THE -- THE DEVICE IS NOT
BEING PURSUED FOR THAT
INDICATION.
BUT THE PERSON OR PERSONS IN THE
TRIAL ARE BENEFITTING FROM IT.
IS THERE ANY WAY THAT THOSE
KINDS OF CARE WOULD BE COVERED?
>> AGAIN I GUESS YOU ASK A VERY
COMPLEX QUESTION.
DEPENDS ON THE FACTS OF THE
MATTER.
BUT WHAT WE DEFINE AS PROVIDING
PATIENTS THE BENEFITS OF PATIENT
WOULD HAVE TO BE BASED ON THE
EVIDENCE.
BASED ON THE RESULTS OF THE
CLINICAL TRIAL.
IT'S NOT JUST THAT IT BENEFITS
THAT PATIENT.
ALL OF THOSE PATIENTS.
FOR THAT INDICATION THAT HAS
BEEN TESTED FOR.
AND SO YOU SAID IT'S SOMETHING
THAT'S BEING PURSUED FOR THAT
INDICATION NOT BEING PURSUED
BEING PURSUED FOR THAT
INDICATION.
NOT THAT THERE'S A WRINKLE IN
IT.
OUR NATIONAL DETERMINATION OF
COVERAGE WHETHER IT'S UNDER CDC
AND IF IT'S A TEST TYPICALLY IF
IT'S AN FDA APPROVED PRODUCT
WE'RE TESTING LOOKING AT THE
EVIDENCE FOR THAT INDICATION.
WE'RE TALKING ABOUT OFF LABEL
INDICATIONS THAT IT'S DIFFICULT
TO ANSWER WITHOUT LOOKING AT THE
FACTS OF THE MATTER.
>> CAN I ASK ONE OTHER WRINKLE
ON THAT.
BETWEEN THE TIME A TRIAL LET'S
SAY A TRIAL IS NOT UNDER CED BUT
IT'S ENDING TAKES A WHILE FOR
FDA APPROVAL, IS THERE ANY
BRIDGE IN THAT INTERSPACE?
>> I WOULD SAY IF WE'RE
COVERING ON THE FRONT END.
DEPENDS ON THE FACTS OF THE
MATTER.
BUT ACTUALLY WE USUALLY LOOK AT
FDA APPROVED DEVICES.
SO THEY'RE APPROVED ALREADY
THEORETICALLY.
BUT TO YOUR QUESTION, IF THE
TRIAL HAS ENDED IS NOT IN THE
CED AND IT TAKES A WHILE FOR US
TO APPROVE, DO WHAT'S CALLED
RECONSIDERATION LATER, BUT
THERE'S -- IF WE HAVE ALREADY
APPROVED IT UNDER THE CED WE
WILL STILL CONTINUE TO COVER IT
IN THE FUTURE.
THAT IS IT DEPENDS ON WHAT IS
THE STATUS OF THE DEVICE IN
TERMS OF APPROVAL FROM OUR END
AT THE TIME IT'S IMPLANTED AND
MAY NOT CHANGE AFTER THAT.
UNTIL WE RECONSIDER THE WHOLE
ISSUE.
THAT'S A SHORT AND DIRTY ANSWER.
OKAY.
>> THAT'S GREAT.
IF YOU DON'T MIND HOLDING YOUR
QUESTION IN WORKING MEMORY FOR A
LITTLE BIT LONGER.
WE'VE GOT THREE MORE
PRESENTATIONS HALFWAY THROUGH
THE PRESENTATIONS WE HAVE
45 MINUTES SCHEDULED AFTER THIS
FOR PANEL DISCUSSION.
IT WILL BE AN EXERCISE IN
WORKING MEMORY FOR ALL OF US
BECAUSE A LOT OF BALLS TO KEEP
UP IN THE AIR IN ORDER TO HAVE A
FULFILLING AND THOROUGH
DISCUSSION AT THE END OF THIS.
I'D LOVE TO INTRODUCE NOW OUR
NEXT SPEAKER, DR. ROBINSON
BEALE.
DR. RHONDA ROBINSON BEALE IS A
SENIOR HEALTHCARE EXECUTIVE
30 YEARS OF EXPERIENCE IN
HEALTHCARE SYSTEMS MANAGED CARE
QUALITY IMPROVEMENT BEHAVIORAL
HEALTH MEDICAL CARE.
SVP DEPUTY CHIEF MEDICAL OFFICER
FOR MENTAL HEALTH SERVICES FOR
UNITED HEALTH GROUP PREVIOUSLY
SERVED AS CHIEF MEDICAL OFFICER
AND PHYSICIAN EXECUTIVE WITHIN
SEVERAL LARGE NATIONAL AND LOCAL
HEALTH ORGANIZATIONS.
OPTUM SUB SUBSIDIARY OF UNITED,
SIGNA AND BLUE CROSS/BLUE SHIELD
MICHIGAN AND HEALTH BLINDS IN
MICHIGAN.
WELCOME DR. BEALE.
WOULD YOU MIND UNMUTING, PLEASE.
>> IF YOU'RE ON A PHONE UNMUTE
BY HITTING STAR 6.
>> HELLO.
>> THERE YOU ARE.
>> THAT'S BETTER NOW, I THINK.
>> OKAY.
ALL RIGHT.
[INAUDIBLE] THE CHALLENGES I'VE
HAD TODAY.
AND FIRST OF ALL, I WANT TO
THANK YOU FOR THE OPPORTUNITY TO
BE HERE FOR THIS IMPORTANT
SUBJECT.
AND IT'S CERTAINLY ONES THAT ALL
THE OTHER PREVIOUS PANELIST
[INAUDIBLE] BEING COMPLEX AND
MULTIFACTORIAL.
AND [INAUDIBLE] VERY, VERY
COMPLEX AND IF IT WAS SIMPLE TO
RESOLVE, IT WOULD HAVE BEEN
RESOLVED BY NOW.
I DO THINK THAT HAVING THIS KIND
OF CONVERSATION WITH
MULTISTAKEHOLDERS AND
[INAUDIBLE] CAN HELP GET
[INAUDIBLE] TO A SOLUTION OR A
DIRECTION OF SOLUTIONS.
I AM NOT ABLE TO SHARE MY SLIDES
WITH YOU GUYS SO I WILL TALK
FROM THEM.
AND TO TALK ABOUT A PAYER'S
PERSPECTIVE.
FIRST OF ALL, TO SAY THAT MY
COMMENTS THAT I'M GOING TO
PRESENT ARE NOT MEANT TO BE A
REPRESENTATIVE OF ANY ONE HEALTH
INSURANCE COMPANY.
I'M REALLY BRINGING THE
EXPERIENCE [INAUDIBLE] TO
INCLUDING UNITED BUT OTHERS IN
TERMS OF [INAUDIBLE] HOW THEY'VE
ADDRESSED THIS PARTICULAR
SUBJECT.
LET'S OVERALL TALK ABOUT
INSURANCE [INAUDIBLE]
[INAUDIBLE] LEVEL SETTING HERE
TO MAKE SURE THAT WE'RE ALL
[INAUDIBLE].
>> EVERY FIFTH WORD OR SO IS
DROPPING OUT.
I WONDER IF THERE'S A WAY TO GET
THE MIC CLOSER.
>> ALL RIGHT.
IS THIS BETTER?
>> I THINK SO.
THANK YOU.
SORRY.
>> OKAY.
SORRY.
SO [INAUDIBLE] HEALTHCARE
INNOVATION DIAGNOSTICS
[INAUDIBLE] MUST BE SUFFICIENTLY
AND CONSISTENTLY [INAUDIBLE] TO
BE IMPACTFUL WITH PATIENT --
THAT'S KIND OF THE BOTTOM IN
TERMS OF THE THRESHOLD THAT
NEEDS TO BE [INAUDIBLE]
COVERAGE.
INSURANCE [INAUDIBLE] THEN
BENEFIT COVERAGE.
[INAUDIBLE] TO INSURANCE
DIFFERENT PAYERS CAN HAVE
DIFFERENT PATHS AS TO WHEN AND
WHAT THEY WILL COVER OF THOSE
TECHNOLOGIES EVEN THOUGH THEY
ARE PROVEN AND EVEN THOUGH
THEY'RE FDA APPROVED.
KEEP IN MIND [INAUDIBLE]
>> DR. ROBINSON BEALE, I'M
SORRY, WONDER IF THERE'S A
DIFFERENT WAY TO CONNECT YOUR
AUDIO.
SEVERAL OF US ARE STILL GETTING
AUDIO DROPOUT.
>> ARE YOU ABLE TO USE AUDIO ON
YOUR COMPUTER, LOOKS LIKE YOU
WEREN'T ABLE TO EARLIER THAT'S
WHY YOU'RE ON YOUR PHONE.
>> THAT'S WHY I CAN'T SEE THE
ICON I TRIED SEVERAL THINGS AS I
WAS SITTING [INAUDIBLE] WOULD
YOU WANT TO GO ON WITH THE
DIFFERENT SPEAKER UNTIL I GET A
DIFFERENT MECHANISM FOR THIS?
>> I THINK THAT'S A GOOD IDEA.
IF YOU DON'T MIND SORRY TO
INCONVENIENCE YOU MAYBE WE CAN
TRY THAT SEE IN THE MEANWHILE IF
WE CAN WORK BACK TO SOME OF THE
OTHER AUDIO OPTIONS.
>> I'LL REACH BACK OUT TO YOU.
>> THANK YOU.
>> WE'LL MOVE FORWARD TO THE
NEXT PANEL SPEAKER AS WE WORK
WITH THE TECH ISSUES WITH DR.
ROBINSON BEALE.
WITH THAT I'LL INTRODUCE SHIRLEY
MCCARTNEY SERVES AS DIRECTOR OF
NEUROLOGICAL SURGERY RESEARCH
FOR BOTH CLINICAL TRIALS AND
INVESTIGATOR INITIATIVE CLINICAL
RESEARCH IN THE SCHOOL OF
MEDICINE AT OREGON HEALTH AND
SCIENCE UNIVERSITY.
THE RESPONSIBILITIES INCLUDE
TEACHING AND DIRECTING FACULTY
AND STAFF OF THE DEPARTMENT OF
NEUROLOGICAL SURGERY ON OHSU
INSTITUTIONAL REVIEW BOARD
POLICIES, CLINICAL TRIAL
MANAGEMENT AND ACADEMIC
PUBLISHING ETHICS.
DR. MCCARTNEY.
>> GOOD AFTERNOON.
CAN YOU HEAR ME?
>> YES, WE CAN HEAR YOU.
>> THANK YOU.
I THINK I MENTIONED EARLIER I
HAVE A DEMENTED HEADSET.
I WOULD MOVE YOU ALL IN FRONT OF
MY NOTES.
I'LL MOVE YOU ALL.
I'D LIKE TO BEGIN THAT THE LAND
FOR OREGON HEALTH SCIENCE RESTS
ON THE TRADITIONAL VILLAGE SITES
OF THE CHINOOK AND OTHER TRIBES
WHO MADE THEIR HOMES AND FORMED
COMMUNITIES ALONG THE COLUMBIA
RIVER.
I'D LIKE TO YOU JOIN ME IN
TAKING THIS OPPORTUNITY TO PAY
RESPECT HONOR AND THANK PAST,
PRESENT AND FUTURE TRIBAL
GENERATIONS FOR THEIR SACRIFICE
AND CARE OF THE LAND.
I DO NOT HAVE ANY DISCLOSURES.
MY THOUGHTS AND COMMENTS TODAY
ARE MY OWN.
I DO NOT REPRESENT THOSE OF
OREGON HEALTH AND SCIENCE
UNIVERSITY.
I TRULY APPRECIATE HEARING FROM
THE PARTICIPANTS THIS MORNING,
IT WAS VERY INFORMATIVE AND VERY
HUMBLING.
AS WE HEARD EARLIER TODAY, WHAT
IS THE BOUNDARY BETWEEN RESEARCH
AND CLINICAL CARE?
AND AT A PREVIOUS MEETING, FOUR
TOPICS WERE HIGHLIGHTED FOR
DISCUSSION AROUND POST-TRIAL
CARE.
AND I'M GOING TO FOCUS MY
DISCUSSION WITH YOU ON THOSE
FOUR TOPICS.
MINIMUM CARE.
SETTING EXPECTATIONS, FACTORS
THAT IMPACT CARE AND COVERAGE
AND GAPS IN CARE.
SO NEXT SLIDE.
MINIMUM CARE.
WHILST TRIAL CARE IS A DIFFICULT
DEFINITION AND CAN BE CONFUSING
TO PATIENTS, WE HAVE HAD
STANDARD OF CARE PATIENTS CALL
OUR CLINK THINKING THEY'RE IN A
CLINICAL TRIAL WHEN IN FACT
THEY'RE NOT.
MANY INSTITUTIONS IMPLANT
DEVICES WITH THE TRIAL OF THE
DEVICE TO DETERMINE EFFICACY
BEFORE PERMANENT DEVICE
IMPLANTATION, AS IS THE CASE FOR
SPINAL CORD STIMULATION.
AND IT'S WORTH REMEMBERING THAT
MANY INSTITUTIONS PROVIDE
STATE-OF-THE-ART POST IMPLANT
DEVICE TRIAL CARE ALREADY FOR
DEVICES THAT ARE APPROVED.
AND ADDED TO THAT, MANUFACTURER
REPRESENTATIVETIVES ARE OFTEN
INVOLVED AND PLAY A PIVOTAL ROLE
IN STANDARD OF CARE.
THE QUESTION IS, DID THE DEVICE
RECEIVE FDA APPROVAL, YES OR NO.
AND THIS ANSWER LIKELY DICTATES
TWO POTENTIAL PATHS FOR MINIMUM
OF CARE.
PATH ONE.
A DEVICE IS APPROVED AND WILL BE
USED COMMERCIALLY.
WAS THERE A PREDETERMINED
THRESHOLD ACROSS STUDIES THAT
DETERMINED BENEFIT?
BENEFIT IN TERMS OF CARE?
WHAT DOES CARE EXTEND TO IN THIS
INSTANCE?
A CLEAR UNDERSTANDING OF BENEFIT
TO THE PATIENTS DOES THIS
INCLUDE CLINICAL BENEFIT ONLY?
IS THE DEVICE PROVIDING CLINICAL
MEASURABLE BENEFIT TO PATIENTS
AND IS THE DEVICE ALSO APPROVED
FOR USE MEDICARE MEDICAID
SERVICES JUST AS WE HEARD.
AND IS THERE ACCESS TO DATA
GENERATED DURING THE CLINICAL
TRIAL PATIENTS PERSONAL DATA AND
GLOBAL DATA AGAIN WE HEARD THAT
EARLIER THIS MORNING.
AND WAS THERE SUFFICIENT TIME
FOR THE PATIENT TO DECIDE IF HE
WISHED TO TRANSITION TO
COMMERCIAL USE OR NOT, THAT IS
TO INSTITUTIONAL STANDARD OF
CARE PROTONS, EVEN IF THOSE ARE
AVAILABLE.
AND I JUST WANT US TO NOT FORGET
AND CONTINUE TO REMEMBER
PATIENTS THEMSELVES PROVIDE THE
DATA THAT IS NEEDED FOR THE
DEVICE APPROVAL.
ETHICALLY, WHO IS REALLY
RESPONSIBLE FOR CONTINUATION OF
CARE IN THIS INSTANCE?
AND PATH TWO, A DEVICE IS NOT
APPROVED.
IF NOT APPROVED, IS THERE AN
AVENUE FOR CONTINUED ACCESS TO
AN INVESTIGATIONAL DEVICE
IF IN A CASE WHERE THERE IS
CLINICAL BENEFIT TO THE PATIENT?
DID THE STUDY INCLUDE COST OF
APPROVAL REPLACEMENT REACHING
END OF LIFE AND ALTERNATE
RESOURCES THAT WERE NOT RELATED
TO THE DEVICE?
WHAT IF THE SPONSOR DECIDES NOT
TO PURSUE FURTHER RESEARCH
AND/OR DEVICE APPROVAL AFTER
STUDY END, WHAT THEN IS THE
SPONSORS ROLE?
AND ONCE AGAIN ETHICALLY WHO IS
RESPONSIBLE FOR THE CARE OF WHAT
COULD NOW BE REFERRED TO AS A
TRIAL ABANDONED PATIENT?
AS ENDORSED HERE WE ALL AGREE
MAINTAINING COORDINATION OF CARE
IS CRITICAL FOR STUDY PATIENTS
LEAVING A DEVICE TRIAL.
NEXT SLIDE.
SETTING EXPECTATIONS FOR ME
PERSONALLY THIS COMES DOWN TO
THREE QUESTIONS, EDUCATION AND
TRUST COMMUNICATION AND
NAVIGATION OF THE HEALTHCARE
SYSTEM ITSELF.
TIME SPENT WITH SUBJECTS IN A
CLINICAL STUDY CAN BE LONG AND
THERE ARE LONG REPEATED VISITS,
VISITS MAY LAST FOR YEARS AND
RELATIONSHIP IS BUILT WITH THE
STUDY TEAM AND THE PRINCIPAL
INVESTIGATOR.
AND SINCE THAT RELATIONSHIP
DEVELOPS OVER TIME, I WONDER IF
THERE'S AN OPPORTUNITY IN THE
INFORMED CONSENT PROCESS TO WORK
ON LANGUAGE, LANGUAGE THAT SETS
EXPECTATIONS RELATED TO
POST-TRIAL CARE, FINANCIAL COST
COULD BE ESTABLISHED DURING
RECRUITMENT AND INFORMED CONSENT
PROCESS, LONG BEFORE THE TRIAL
IS EVEN COMPLETED.
FOR SOME CLINICAL TRIALS THE
INFORMED CONSENT PROCESS
INVOLVES PROVIDING THE PATIENTS
WITH FINANCIAL INFORMATION ABOUT
THE COST OF COVERAGE THROUGHOUT
THE TRIAL BUT NOT NECESSARILY
AFTER THE TRIAL HAS ENDED AND
WE'VE HEARD THAT ALREADY TODAY
ALSO.
WHAT I'M SUGGESTING IS TO
PROVIDE PATIENTS WITH
INFORMATION RELATED TO POSSIBLE
FINANCIAL BURDEN OR NOT THAT
MIGHT OCCUR AT A LATER DATE LONG
BEFORE THEY AGREE TO
PARTICIPATE.
HOWEVER, THAT BEING SAID,
FORECASTING THE POSSIBLE NEEDS
OF A PATIENT WITH A DEVICE SUCH
AS A BATTERY REPLACEMENT OR A
NEED
OR COMPATIBILITY AS WE HEARD
EARLIER TODAY CAN ALSO BE
DIFFICULT.
AND PATIENTS MAY STILL NEED
ADDITIONAL SURGERY.
WITH INPUT FROM SOME OF MY
NEUROSURGICAL COLLEAGUES.
HAVE TWO EXAMPLES OF TRIAL
PATIENTS, WHERE SETTING
EXPECTATIONS EARLY ON MAY HAVE
HELPED.
IN A STUDY FOR DEEP BRAIN
STIMULATION FOR DEPRESSION,
SPONSOR, PAYING FOR GENERATOR
REPLACEMENTS, AND THE PATIENTS
WERE LEFT WITH NO RECOURSE.
AFTER THE TRIAL WAS CLOSED AND
THE RENEWAL INSURANCE COVERAGE,
GANGLING STIMULATION PER CLUSTER
THE SPONSOR FILED FOR BANKRUPTCY
AND THE UNIVERSITY FOUND
THEMSELVES IN A POSITION THAT
THEY HAD TO FIND RESOURCES TO
COVER A IMPLEMENTATION OF THE
DEVICE. ETHICALLY, WHO'S
RESPONSIBLE FOR THE CONTINUATION
OF CARE, AND THE CONTINUATION OF
COVERING COST.
NEST SLIDE.
FACTORS THAT IMPACT COVERAGE.
THE HEALTH CARE SYSTEM ITSELF.
MEDICALLY UNDER SERVED
COMMUNITIES AND POPULATIONS JUST
GENERALLY DO NOT HAVE ACCESS.
TRUST.
IF THE PANDEMIC HAS TAUGHT US
ANYTHING, THAT IS THERE IS A
LACK OF TRUST IN MANY
COMMUNITIES RELATED TO HEALTH
CARE ISSUES.
STATE AND FEDERAL LIMITATIONS,
AND I HAVE TWO EXAMPLES, I CAN
TELL YOU THAT IN OREGON PSYCHO
SURGERY IS AMONG THE LIST OF
CONDUCT THAT IS GROUND FOR
DISCIPLINE.
PSYCHOSURGERY IS DEFINED AS ANY
OPERATION DESIGNED TO PRODUCE AN
IRREVERSIBLE LESION OR DESTROY
BRAIN TISSUE FOR THE PRIMARY
PURPOSE OF ALTERING THE
THOUGHTS, EMOTIONS, OR BEHAV
BEHAVIOROF A HUMAN BEING.
HOWEVER THE TERM DOES NOT
INCLUDE PROCEDURES UNDER TAKEN
TO CURE WELL DEFINED DISEASE
STATES, SUCH AS BRAIN TUMOR
EPILEPTIC FOCI AND CERTAIN
CHRONIC PAIN SYNDROMES.IN
ADDITION SHE NOTIC BRAIN SURGERY
FOR OTHER PURPOSES, ALSO
ACCEPTABLE. SO SOME OF YOU WHO
ARE IN THE PANEL THIS MORNING
WOULD NOT BE HAVE ACCESS TO THIS
PROCEDURE, IN OREGON.
HIGHLY EX-PER EXPERIENCEDED
TEAM PERFORMING THE FIRST DEEP
BRAIN SURGERY FOR PARKINSON'S
DISEASE IN THE UNITED STATES,
IN 1991, AND THIS HAS EXPERTISE
IN OUR STATE, THAT CANNOT BE
UTILIZED AND THIS IS A LOST
OPPORTUNITY. ALSO ADD THE OREGON
IS A VERY INTERESTING STATE. WE
DO HAVE REALIZED PSILOCYBIN FOR
TREATMENT OF DEPRESSION. SO IT'S
AN INTERESTING PLACE TO LIVE.
PERIPHERAL NERVE STIMULATION WAS
INVENTED IN THE 1960S, EVEN
BEFORE SPINAL CORD STIMULATION,
STARTING IN 2012 A NUMBER OF
THERAPEUTIC DEVICES THAT
STIMULATE PERIPHERAL NERVES OR
THAT PROVIDE PERIPHERAL NERVE
FIELD STIMULATION RECEIVED WITH
THE APPROVAL IN A NUMBER OF
REGIONS AROUND THE WORLD FOR THE
TREATMENT OF NEUROPATHIC PAIN,
AND, AND SOME GEOGRAPHIC AREAS
MIGRAINE HEADACHES AND
OVERACTIVE BLADDER, BUT NOT IN
ALL GEOGRAPHIC AREAS.
AGAIN, THIS COMES DOWN TO
ACCESS.
GEOGRAPHY CAN BE A CHALLENGE.
SOME RESEARCH STUDIES INCLUDE
TRAVEL COMPENSATION FOR
SUBJECTS.
ONE ASSUMES THAT THIS WOULD NO
LONGER BE AVAILABLE IF A DEVICE
IS SUBSEQUENTLY APPROVED FOR
USE. AND THEN WHAT THAT MEANS
THERE'S LOCAL ACCESS TO CARE AND
INDIVIDUALS WHO CAN HELP
COORDINATE OR SUPPORT THE
TRANSITION FROM CLINICAL
RESEARCH TRIAL PARTICIPANTS TO
STANDARD OF CARE JUST MAY NOT BE
AVAILABLE. FINANCIAL CONCERNS
AND ADEQUATE INSURANCE COVERAGE
IS LIKELY IN MOST CASES AT THE
COST OF THE INVESTIGATIONAL
DEVICE AND CARE WERE COVERED
WHILE THE PAYMENT IN THE STUDY
AND AN ACTIVE PARTICIPANT. AS
REFERENCED EARLIER, DE
PRIORITIZED BY THE FUNDING
ENTITYPATIENTS MAY BE LEFT
WITHOUT SUPPORT. THAT IS THE
BECOME TRIAL ABANDONED.
THE TYPE OF SPONSOR, IS IT
FEDERAL OR INDUSTRY SPONSOR, I
WONDER ALSO IF THERE IS AN
OPPORTUNITY TO CONSIDER LANGUAGE
AND CONTRACTS WITH REGARDS TO
THE LEVEL OF FUNDING FROM THE
ENTY IF THEY DO NOT COVER COST,
PARTICULARLY THOSE WITH DEVICE
MAINTENANCE.
AND GAPS.
MAINTAINING COORDINATION OF CARE
IS CRITICAL FOR STUDY
PARTICIPANTS LEAVING A TRIAL.
PERHAPS NIH PARTNERSHIP WITH
CENTERS FOR MEDICARE AND
MEDICAID SERVICES, AND SPONSORS
COULD WORK TO PROVIDE RESOURCES
FOR SUCH SITUATIONS, AND IT
SOUNDS LIKE THAT'S HAPPENED IN
SOME SITUATIONS AS IT WAS
DISCUSSED FOR NEUROCASE.
FUNDING FOR POST TRAIL CARE FOR
ALL PARTICIPANTS, A MINIMUM WAGE
FOR INVESTIGATIONAL DEVICE
REMOVAL SHOULD THE PATIENT
DESIRE THAT. A SOLUTION COULD BE
EMPLOYED TO A SOLUTION COULD BE
TO EMPLOY PATIENT ADVOCATES,
MUCH LIKE COORDINATORS WHO SERVE
AS LIAISONS BETWEEN THE CLINICAL
TRIAL TEAM, AND PATIENTS
THROUGHOUT THE PATIENT'S LIFE.
TO ENSURE THAT ONE, ARE IS
PROPERLY COORDINATED. TWO, NEW
FINDINGS FROM CLINICAL TRIAL
ANALYSIS ARE SHARED WITH THE
PATIENTS AND THREE: PATIENTS
CAN OBTAIN SUPPORT FOR THEIR
IMPLANTED DEVICES, AND WE COULD
THINK OF THIS AS SIMILAR TO THE
MANUFACTURER REPRESENTATIVES WHO
ARE OFTEN INVOLVED IN STANDARD
OF CARE, POST DEVICE. HOWEVER,
IN MOST CASES, ACCESS TO SUPPORT
AND RESOURCES, EG. FOR EXAMPLE,
SOCIAL WORKERS OR STUDY
COORDINATORS MAY BE LIMITED TO
THE PERIOD DIRECTLY FOLLOWING
CLINICAL TRIAL INVOLVEMENT,
RATHER THAN AN EXTENDED
THROUGHOUT THE PATIENT'S
LIFETIME, EAVING PATIENTS WITH
NO LONG TERM SUPPORT TO
COORDINATE CARE AND ABILITY TO
NAVIGATE INSURANCE ISSUES.
IT'S IMPERATIVE THAT SOLUTION BE
IDENTIFIED TO ENSURE NO GAPS
BETWEEN PATIENT CARE
COORDINATION AND SUPPORT, THANK
YOU.
I REALLY LOOK FORWARD TO
DISCUSSING OPPORTUNITIES WHERE
GROWTH AND DEVELOPMENT MIGHT BE.
DOES ANYONE HAVE ANY QUESTIONS?
WE HAVE TIME FOR QUESTIONS, IF
ANYONE HAS ONE.
>> I CAN ASK A QUESTION.
SO THIS WAS REALLY HELPFUL.
THANK YOU FOR YOUR INSIGHTS.
I WAS WONDERING IF YOU COULD
GIVE SOME SORT OF INSIGHTS INTO
WHAT FROM AN INSTITUTIONAL
PERSPECTIVE IS SORT OF --
SOMETIMES PROVIDED, ALWAYS
PROVIDED, NEVER PROVIDED WHEN IT
COMES TO POST-TRIAL CARE.
>> IT'S REALLY, REALLY HARD TO
NAVIGATE EVEN WITHIN A STUDY.
AND I WOULD PUSH BACK TO A
COMMENT A LITTLE BIT EARLIER, MY
EXPERIENCE FROM RESEARCH
ADMINISTRATION STANDPOINT, WE DO
NOT MAKE MONEY OUT OF STUDIES
AND BARELY BREAK EVEN, IN MANY
CASES WE LOSE FUNDING, ON THE
PLUS SIDE THE PATIENTS DO GET TO
BILLED UP A REALLY CLOSE
RELATIONSHIP WITH PROVIDERS.
IN THIS CASE THE STUDY
COORDINATORS.
AND ONE THING I HAVE BEEN
THINKING ABOUT ALSO IN A BIGGER
SENSE IS THAT NO ONE REALLY
OFFERS CLINICAL RESEARCH
COORDINATOR EXPERIENCE AS A
CAREER.
AND I DO SEE THAT NOW AS BEING A
CAREER.
BECAUSE, THEY ARE DEFINITELY THE
PEOPLE WHO CAN BEST NAVIGATE
WHAT'S IN THE CONSENT FORM, IN
THE CONTRACT, AND WORK REALLY
CLOSELY WITH PATIENTS FOR THEIR
BENEFIT.
BUT NAVIGATING WHAT IS PROVIDED,
AND NOT PROVIDED IS REALLY,
REALLY DIFFICULT FOR THE
COORDINATORS OFTEN.
>> THANK YOU.
>> DR. ROBINSON BEALE, LET'S
CHECK IN AGAIN IF YOU HAD A
CHANCE WITH A DIFFERENT AUDIO
STRATEGY.
>> ALL RIGHT, CAN YOU HEAR ME
THIS TIME?
>> YES, YOU SOUND WAY BETTER.
>> VERY PROMISING.
>> PERFECT.
>> ALL RIGHT, GREAT.
THANK YOU SO MUCH.
SAMEER FOR YOUR GUIDANCE ON
THAT.
I WAS VERY PANICKED BY THIS, I
WANT YOU TO KNOW.
LET ME GO BACK AGAIN AND QUICKLY
SAY THAT WHAT I'M GOING TO
PRESENT IS NOT REPRESENTATIVE OF
ANY ONE INSURANCE.
WHAT I DID IN PREPARATION WAS
TALK TO MANY COLLEAGUES FROM
DIFFERENT INSURERS TO GET A
COMMON THEME AS TO HOW THEY
APPROACH THESE ISSUES AND WHAT
ARE THE ISSUES AND CONCERNS THAT
THEY HAVE.
AS AN OVERVIEW, IT'S REAL
IMPORTANT TO UNDERSTAND THAT THE
GENERAL THRESHOLD FOR COVERAGE
BY INSURERS IS THAT THE
TECHNOLOGY WHETHER FOR
DIAGNOSTIC TREATMENTS AND
DEVICES AND OTHERS, MUST BE
SUFFICIENTLY AND CONSISTENTLY
RESEARCH-PROVEN, AND NEEDS TO
SHOW IT HAS IMPACT, AND
EFFICACIOUS AND PROVIDES PATIENT
SAFETY.
THIS IS FOR INSURANCE COVERAGE
THAT IS DIFFERENT THAN A
BENEFIT.
MEANING THAT THERE IS SPECIFIC
INFORMATION WITHIN THE INSURANCE
CONTRACT THAT SAYS THAT THIS
PARTICULAR TYPE OF SERVICE IS
SOMETHING THAT IS COVERED.
IN MOST CASES THE THE GENERAL
ACCEPTED TREATMENTS ARE, BUT
THERE ARE SOME EXCEPTIONS THAT
CAN POP UP.
THE REASON WHY I BRING THIS UP
IS BECAUSE WHEN YOU LOOK AT
PAIRS THERE ARE DIFFERENT TYPES
OF PAYERS.
YOU HAVE EMPLOYERS WHO FUND
MAJORITY OF HEALTH CARE,
MEDICARE IS NEXT MEDICAID IS
NEXT AND AFTER THAT MEDICAID
CHIP AND AFFORDABLE CARE ACT.
THERE ARE ALSO THOSE
PROVIDERS -- LARGE SYSTEMS THAT
ARE CAPITATED.
THEY DON'T NECESSARILY CREATE
THEIR OWN INSURANCE BENEFITS AND
GENERAL COVER THE INSURANCE
COVERAGE, THE SCOPE OF SERVICES
COVERED BY THE INSURER THAT SET
THEM UP IN CAPITATED
ARRANGEMENT, AND THEN LAST
CATEGORY OF PAYERS ARE
INDIVIDUALS WHO PAY FOR THEIR
OWN, AND THAT'S PORTAL OF DIRECT
TO CONSUMER TYPE OF CARE, AND
MANY TIMES THOSE ARE CARE OR
SERVICES THAT HAVE NOT BEEN
PROVEN TO BE THROUGH RESEARCH.
AND WHAT I WOULD CALL THE
GROUND-UP TYPE OF RESEARCH THAT
IS DONE BY PROVIDERS WHO ARE
FINDING NEW WAYS TO APPLY
EXISTING TECHNOLOGIES AND MANY
TIMES ARE FINDING WITH
INDIVIDUAL PATIENTS SOME
POSITIVE EFFECT.
I BRING THAT UP, BECAUSE THAT
IS A WHOLE DIFFERENT ROAD TO
INSURANCE COVERAGE AS OPPOSED TO
THOSE COMING THROUGH THE TYPICAL
RO
ROUTE OF CLINICAL TRIALS AND
RESEARCH, AND OTHER
METHODOLOGIES.
THE FACTORS THAT IMPACT
INSURANCE COVERAGE ARE THE
DIFFERENCE IN THE PAYER SOURCES
AS I MENTIONED.
POLICY VERSUS BENEFIT WHICH I
JUST MENTIONED AND THE DIVE
DIVERGENCE OF COST OFFSET.
THAT HAS BEEN ALLUDED TO BY MANY
OF THE PANELISTS THAT ON THE
MANUFACTURERS SIDE BUT ALSO ON
THE INSURANCE COVERAGE SIGN.
THERE IS A CONSIDERATION,
CERTAINLY NOT THE ONLY
CONSIDERATION, BUT IT IS A
CONSIDERATION IN LOOKING AT
COVERAGE IS THAT, OF HAVING A
COST OFFSET. REASON BEING IS IF
YOU LOOK AT INSURANCE OR
PAYERS -- I WON'T SAY BANKERS --
BUT THE ADMINISTRATOR OVER A
TRUST.
AND THE TRUST HAS A CERTAIN
AMOUNT OF MONEY IN IT.
AND IT IS TO BE APPLIED TO THE
POPULATION IT SERVES.
SO PART OF THE DECISION-MAKING
IS AS NEW TECHNOLOGIES COME IN,
HOW DO YOU FIT THAT WITHIN THE
CERTAIN AMOUNT OF MONEY THAT IS
THERE TO BE DISTRIBUTED TO THE
POPULATION THAT WOULD BE
UTILIZING SERVICES.
WE KNOW NOW THAT 5-7% OF THE
POPULATION CONSUMES 82% OF THE
DOLLARS, FOR MEDICAL COVERAGE.
RECOGNIZING THIS DIFFERENCE IT
IS VERY IMPORTANT TO UNDERSTAND
WHEN SOMETHING IS PROVEN
EFFICACIOUS AND MANY OTHER
QUESTIONS THAT NEED TO BE ASKED.
I THINK DR. LI STARTED SOME OF
THOSE, AND I'LL JUST ADD TO SOME
OF THOSE QUESTIONS.
IT'S NOT ONLY LOOKING AT WHETHER
IT'S EFFICACIOUS BUT HOW LONG IS
IT EFFICACIOUS?
DOES IT NEED TO BE REPEATED?
WHAT'S THE LONG TERM
RAMIFICATIONS OF USING THIS
PARTICULAR TREATMENT, OR THIS
DIAGNOSTIC SO THAT ONE BEGINS TO
UNDERSTAND THIS THIS PERSON
GOING TO NEED TO HAVE ONGOING
CARE, WE TALKED ABOUT
REPLACEMENT REPEAT SURGERY, ALL
OF THOSE PIECES NEED TO BE TAKEN
IN CONSIDERATION AS WELL AS
IDENTIFIED IN ANSWERED TO GIVE A
FULL SCOPE OF WHAT THIS
PARTICULAR PROCEDURE IN THE LONG
RUN IS GOING TO CAUSE AND HOW IT
FITS INTO THIS GENERAL POT THAT
I'VE DESCRIBED. THAT IS THE
GENERAL POT OF MONEY THAT IS
USED FOR THE TOTAL POPULATION.
THE REASON WHY THAT IS IMPORTANT
AND AS WE ALL ARE AWARE IN
UNITED STATES HEALTH CARE IS
EXTREMELY EXPENSIVE.
AND OVERALL OUTCOME AND
COMPARISON TO OTHER COUNTRIES IS
VERY POOR.
BUT ALSO EXPERIENCING CONTINUING
EXPERIENCE OF ESCALATING HEALTH
CARE COSTS THAT GETS PASSED ON
TO INDIVIDUALS TERMS OF PREMIUMS
AND COPAYMENTS AND COINSURANCE
ON A WAY TO TRY AND CURB SOME OF
THAT COST.
IN TERMS OF LOOKING AT THOSE
TECHNOLOGIES AND WHETHER OR NOT
COVERED.
DR. LI MENTIONED THE WHOLE
EVIDENCE -- THE HIERARCHY OF
EVIDENCE USED.
METAANALYSIS, RANDOMIZED
CLINICAL TRIALS, AND THOSE IN
LARGE RANDOMIZED TRIALS.
WHAT IS REALLY IMPORTANT IN MOST
HEALTH PLANS, THERE IS A POLICY
COMMITTEE THAT LOOKS AT THE
EVIDENCE, REVIEWS THE EVIDENCE
AND DETERMINES WHETHER OR NOT
THE EVIDENCE THAT HAS BEEN
PRESENTEDED DEMONSTRATES EFFECT
EFFICACIOUSNESS IN TERMS OF A
PATIENT POPULATION. HOPEFULLY
THAT IT'S SPECIFIC ENOUGH THAT
ONE CAN IDENTIFY THE PATIENTS
THAT PARTICULAR TECHNOLOGY WILL
WORK BEST FOR AND IDENTIFY WHO
IS WHAT TYPE OF TRAINING IS
NECESSARY FOR THAT TECHNOLOGY TO
BE RENDERED, AND RENDERED WELL
WITH FIDELITY.
AS FELL, AS I MENTIONED BEFORE,
HOW LONG DOES TREATMENT OCCUR.
. WHAT'S THE LONG TERM EFFECT,
ALL OF THOSE PIECES. EVEN IF THE
EVIDENCE IS NOT PRESENT, AND
PARTICULARLY IF IT'S A CONDITION
THAT THERE IS THE POSSIBILITY OF
IMMINENT DEATH. THERE ARE
REGULATIONS THAT REALLY REQUIRE
PAYERS TO COVER NONPROVEN
TECHNOLOGIES WITH A CERTAIN
LEVEL OF RIGOR IN THOSE
TECHNOLOGIES, AND MOST COMMONLY
APPLIED TO CANCER.
THE OTHER AREAS THAT ARE USED
TO DETERMINE THINGS THAT ARE
COVERED ARE THOSE THAT ARE
CONSIDERED WIDELY ACCEPTED
MEDICAL PRACTICES, AND GENERALLY
THOSE ARE THINGS THAT HAVE BEEN
LONG STANDING AND HAVE NOT
NECESSARILY GONE THROUGH THE
RIGOR OF RESEARCH, FORMAL
RESEARCH, BUT HAS BEEN VETTED
THROUGH THE WIDE POPULATION
EXPOSURE TO THOSE PARTICULAR
SERVICES.
OTHERS ARE PROVIDED
ORGANIZATIONS THAT GENERATE
EVIDENCE-BASED GUIDELINES THAT
IS COMBINATION OF RESEARCH, AND
ALSO PRACTICE WHERE THEY BRING
IN EXPERIENCE OF PROVIDERS
RENDERING THE SERVICES AND HELP
TO COMBINE THAT INFORMATION
TOGETHER TO DEVELOP GUIDELINES,
PRACTICAL GUIDELINES.
AND THEN NATIONAL CONSENSUS
STATEMENTS AND PUBLICATIONS THAT
ARE USED.
>> I THINK WE LOST THE
CONNECTION.
>>OUR LAST SPEAKER OF THE
SESSION, A PLEASURE TO INTRODUCE
IS DR. LANGHALS.
WHO SERVES AS SUPERVISORY
PROGRAM DIRECTOR, LEADING THE
TRANSLATIONAL DEVICES TEAM WITH
WITHIN THE DIVISION OF
TRANSLATIONAL RESEARCH E IS A
TEAM LEAD IN BOTH THE UNITED
BRAIN INITIATIVE, AS WELL AS THE
BLUEPRINT MED TECH PROGRAM AND
HIS TEAM ALSO LEADS EFFORTS
WITHIN THE STIMULATING
PERIPHERAL ACTIVITY TO RELIEVE
CONDITIONS SPARK PROGRAM, AND
LONG TERM THE HEAL INITIATIVE.
CURRENTLY OVERSEES THE GRANT
PORTFOLIO IN THE AREAS OF NEURO
TECHNOLOGY DEVELOPMENT
VALIDATION AND TRANSLATION FOR
APPLICATIONS IN NEUROSCIENCE AND
PHYSIOLOGY MOVING DISORDERS. DR.
LANGHALS.
>> I'LL JUST WALK THROUGH
LITTLE BIT OF THE NIH SIDE OF
THINGS.
ND WHAT I'VE DONE AND WE'VE
DONE WITHIN SEVERAL OF OUR
TRANSLATION WILL PROGRAMS AS IT
RELATES TO LOOKING AT CONTINUING
TRIAL RESPONSIBILITIES AND
POST-TRIAL CARE.
NEXT SLIDE.
SO OVERALL IN OUR PROGRAMS, AND
THE BRAND INITIATIVE IN
PARTICULAR, WE SUPPORT VARIOUS
DIFFERENT FUNDING OPPORTUNITIES
FROM VERY EARLY STAGE AND OF
CONCEPT GENERATION AND DEVICE
DEVELOPMENT ALL THE WAY THROUGH
LOTS OF THE THESE ACTIVITIES IN
FIRST IN HUMAN TRIALS OF NEW
DEVICES, EARLY FEASIBILITY
STUDIES AND AN ADDITIONAL
ACTIVITIES IN THE CLINICAL
TRIALS PIPELINE. IN PARTICULAR,
AS NOTED HERE, WITH WITHIN MY
GROUP WE'RE MANAGING ACTIVITIES
BOTH WITHIN THESE BRAIN
INITIATIVE PROGRAMS IN THE
SUPPORT BOTH THE LATE STAGE
PRECLINICAL DEVELOPMENT. AND
FIRST IN HUMAN TRIALS IN THE
PHASE 2 AWARD.
AND AS WELL AS APPLICATION FOR
DIRECT TO UH3'S, FOR THOSE READY
TO BEGIN THE CLINICAL TRIAL OF
THE TECHNOLOGY IN NEW TARGET OR
NEW INDICATION.
AND SIMILARLY, WE SYNERGIZED A
LOT OF THE LANGUAGE IN OUR
FUNDING OPPORTUNITIES ACROSS THE
NEWLY LAUNCHED BLUEPRINT MED
TECH PROGRAM THAT IS DESIGNED
AROUND FACILITATING SOME OF
THESE ACTIVITIES AND PROGRAMS
THAT ALSO SUPPORT THESE AREAS OF
TECHNOLOGY DEVELOPMENT.
NEXT SLIDE.
SO KIND OF THE MAIN DIFFERENCES
AS I MENTIONED, THESE PROGRAMS
ARE TO SUPPORT THESE TECHNOLOGY
DEVELOPMENT ACTIVITIES. PROGRAM
SUPPORT THOSE WITHIN OUR
MISSION. HOWEVER FOR THE BRAIN
INITIATIVE PROGRAMS. THERE ARE
10 PARTICIPATING INSTITUTES AND
CENTERS THAT WE ARE MANAGING
THESE VARIOUS DIFFERENT
TRANSLATIONAL ACTIVITIES ON
BEHALF OF THE FED IN THE AREA OF
INVASIVE NEURAL DEVICES FOR BOTH
RECORDING AND STIMULATING TO
TREAT AND DIAGNOSE DISORDERS OF
THE CENTRAL NERVOUS SYSTEM AS
WELL AS TO ACCOMPLISH THE GOALS
OF THE BRAIN INITIATIVE WHICH
INCLUDING TO BETTER UNDERSTAND
THE CIRCUITS WITHIN THE BRAIN.
AND THE SECONDARY GOAL OF OUR
PROGRAMS IN THE BRAIN INITIATIVE
OR FACILITATE THESE PUBLIC
PRIVATE PARTNERSHIPS BETWEEN
INVESTIGATORS AND MANUFACTURERS
HAVE THE LATEST GENERATION WITH
THE STIMULATING AND RECORDING
DEVICES. AND SIMILARLY WITHIN
THE BLUE PRINT MEDICINE TECH
PROGRAM IS DESIGNED TO NOT ONLY
PROVIDE FUNDING FOR SUPPORTING
THESE ACTIVITY, BUT ALSO SETTING
UP VARIOUS CONTRACT AND
CONSULTAN
CONSULTANTSY RESOURCES AND
INCUBATOR HUBS TO HELP THE
INVESTIGATORS WORK AT ALONG THIS
DEVELOPMENT PIPELINE FOR
BRINGING NEW TECHNOLOGIES AND
NOVEL TECHNOLOGIES ALONG THAT
PIPELINE TOWARDS DE RISKING THEM
FOR THE FIRST IN HUMAN
DEMONSTRATION IN THE BRAIN
INITIATIVE HAS HAS SIGNED ON TO
THAT BLUEPRINT MED TECH PROGRAM
AS WELL TO POTENTIALLY SUPPORT
ACTIVITIES WITHIN THE BRAIN
INITIATIVE'S MISSION. .
NEXT SLIDE.
IN TERMS OF OVERALL PUBLIC
PRIVATE PARTNERSHIP PROGRAM, ONE
OF THE THINGS TO NOTE IS THAT IN
GENERAL NIH IS SERVING A ROLE TO
HELP FACILITATE THESE
PARTNERSHIPS, BUT NOT
NECESSARILY A PARTY FROM THE
LEGAL PERSPECTIVE TO THE
INDIVIDUAL AGREEMENT.
SO YOU CAN SEE THE GENERAL
PROCESS, WHEREBY THE APPLICANT,
THE ACADEMIC PI IDENTIFIES THE
COMPANY COLLABORATOR, VICE VERSA
AND SIGN THEIR OWN CONFLICT
DISCLOSURE AGREEMENT BETWEEN THE
VARIOUS ORGANIZATIONS.
AND THEN CAN USE THAT
PARTNERSHIP TO SUBMIT
APPLICATION TO EITHER THESE
BRAIN UG3 OR UH3 OR DIRECT UHV
FUNDING OPPORTUNITIES THAT
INCLUDE A LETTER OF SUPPORT FROM
THE COMPANY.
AND FOR APPLICATIONS THAT SCORE
WELL AND LOOKING AT PROPOSING
FOR FUNDING, WE DO REQUIRE THAT
THE ACADEMIC INVESTIGATOR,
AND/OR INVESTIGATORS AS WELL AS
THE COMPANY SIGN A COLLABORATIVE
RESEARCH AGREEMENT OR CLINICAL
TRIALS AGREEMENT PRIOR TO THE
NOTICE OF GRAND AWARD.
HOWEVER WE CAN NOT DICTATE THE
TERMS OF THOSE AGREEMENTS
BETWEEN THE VARIOUS DIFFERENT
PARTIES.
SO AS PART OF OUR CURRENT PUBLIC
PRIVATE PARTNERSHIP ACTIVITIES
WE HAVE SEVERAL DIFFERENT
COMPANIES THAT HAVE SIGNED ON
WITH MOU, MEMORANDUM OF
UNDERSTANDING WITH THE NIH TO
SHARE INFORMATION ABOUT THEIR
TECHNOLOGIES, AND YOU CAN SEE
THE LIST OF COMPANIES CURRENTLY
PARTICIPATING ON THE PLOT HERE,
AND NOTE SEVERAL OF THE
COMPANIES YOU HEARD ABOUT TODAY
ARE ACTIVELY PARTICIPATING.
NEXT SLIDE.
SO, AS PART OF THE GRANT
APPLICATIONS, IT WAS MENTIONED
EARLIER TODAY, WE DO HAVE THE
ABILITY TO SET UP LONG TERM CARE
PLAN ATTACHMENTS AS PART OF OUR
FUNDING OPPORTUNITIES AND WITH
EACH ONE OF MY PROGRAMS WITHIN
THE BRAIN INITIATIVE.
WE HAVE MADE THIS REQUIREMENT
FOR APPLICANTS COMING INTO THE
FUNDING OPPORTUNITY, AND
SEPARATE ATTACHMENT, SO DOESN'T
TAKE AWAY FROM THE PAGE LIMITS
OF THE REST OF THE APPLICATION.
AND THEY CAN HAVE INSTRUCTIONS
AS PART OF THE FUNDING
OPPORTUNITIES, DICTATING THE
APPLICANTS REALLY SHOULD BE
DESCRIBING THE ANTICIPATED CARE
NEEDS AS IT RELATES TO STUDIES,
AND LOOKING AT APPLICATIONS TO
POST-TRIAL SCENARIOS AND WHETHER
THE DEVICE IS TRIAL OR FAILURE
OR SUCCESS.
AND BASED ON THE PREDESCRIBED
OUTCOMES, AND AS WELL WHAT MAY
HAPPEN IN THE DEVICE
MANUFACTURES DISCONTINUE THE
PRODUCT OR FAIL TO COMMERCIALIZE
IT.
IN PARTICULAR THE APPLICANTS
SHOULD REALLY BE LOOKING AT END
OF TRIAL STUDY PLANS, AND
VARIOUS DIFFERENT SITUATIONS
RELATED TO THE RISKS AND
BENEFITS OF RECEIVING THE CARE
OR NOT RECEIVING THE CARE.
AND PROVIDE SOME EXAMPLES OF THE
THINGS THAT THE PLANS MAY
INCLUDE THAT YOU HEARD ABOUT
EARLIER TODAY INCLUDING PLAN TO
THE DEVICES.
ONCE IT'S COMPLETE.
POTENTIAL SURGICAL REMOVAL OF
THE BATTERY AND CAPPING THE
LEADS TO MINIMIZE SOME OF THE
RISKS, AS NOTED SOME
MANUFACTURERS HAVE OFFERED TO
PROVIDE THE DEVICE RELATED
MAINTENANCE FOR PATIENTS
RESPONDING TO THE THERAPY, AND
AS WELL AS MANUFACTURE SUPPORT
FOR FILING COMPASSIONATE USE
EXEMPTIONS OR OTHER ACTIVITIES
TO HELP SUPPORT THOSE AREAS. AND
THEN ALL THE PLANS SHOULD REALLY
INCLUDE THOSE POST TRIAL
OBLIGATIONS AS WELL AS CAN
BUDGET FOR THE EXPENSES
ASSOCIATED WITH THAT GRANT
APPLICATION.
NEXT SLIDE.
SO COMMON ELEMENTS WE HAVE SEEN
OVER THE YEARS WITH VARIOUS
DIFFERENT ACTIVITIES.
AS MENTIONED THE DEVICE REMOVAL
AT THE END OF THE STUDY, THAT
CAN BE COVERED BY THE GRANT
FUNDS, POTENTIALLY DEVICE
REPLACEMENT AND SUBSEQUENT
PATIENT MONITORING, AND
POTENTIALLY ABLE TO BE COVERED
THROUGH INSURANCE MECHANISMS AND
GRANT FUNDS AS WELL.
THERE IS THE POSSIBILITY OF SOME
OF THEM PROPOSING CONTINUEDED
USE AFTER THE STUDY HAS ENDED.
BUT HAVING PLANS FOR REPAIRING
THE EXISTING HARDWARE AND
LOOKING AT ON GOING MONITORING,
AND MEDICAL SUPPORT ON GOING
COVERAGE, OR POTENTIALLY-TIME
HOSPITAL CARE.
SOME HAVE PROPOSED THE PURCHASE
DESIGNATED PRIVATE INSURANCE
PLAN SPECIFIC TO THOSE POST
TRIAL NEEDS. AS WE HEARD ABOUT
EARLIER TODAY, WE'VE SEEN THAT
THAT SUCCESSFULLY AND FROM
IMPLEMENTED OVERSEAS, BUT NOT SO
FAR IN THE US.
AND MOST OF THE PROPOSALS GIVE
THE PATIENTS THE OPTION TO
CONSIDER AT THE END OF THE STUDY
TO EITHER CONTINUE WITHIN ONE OF
THE VARIOUS DIFFERENT STRATEGIES
AND KEEP THE INVESTIGATIONAL
DEVICE EXEMPTION OPEN TO HELP
SUPPORT AND THOSE KIND OF LONGER
TERM FOLLOW UP TO, FOR THE
PATIENTS. IN SOME OF THE
INDICATIONS WHERE WE'RE
SUPPORTING PROJECTS THERE MAY BE
ALTERNATIVE TO CHANGE TO
COMMERCIALLY AVAILABLE DEVICE,
SO THERE AREN'T AS MANY CONCERNS
WITH THAT ONGOING CARE OF
RESEARCH DEVICE THAT'S BEEN
IMPLANTED AS PART OF THE STUDY.
IN SOME CASES THEY'RE GIVEN THE
OPTION TO 0 OUT THE DEVICE AND
TURN IT OFF, OR LEAVE IN PLACE.
OR AS MENTIONED THE EXPLANTATION
OPTION.
AND IN SOME CASES HOSPITALS HAVE
BEEN WILLING TO PROVIDE THAT
CARE IN PERPETUITY.
IN OTHER CASES THEY HAVE BEEN
LESS ENTHUSIASTIC OF THOSE TYPES
OF ACTIVITIES.
NEXT SLIDE.
I WAS GOING TO TALK A LITTLE
BIT ABOUT CMS OPTIONS, BUT WE
COVERED THOSE EARLIER, AND GO ON
TO THE NEXT SLIDE, NOW.
AND SO WHEN LOOKING AT MANAGING
THESE TYPES OF PROJECTS FROM A
PROGRAMMATIC STANDPOINT WE DO
HAVE SOME TOOLS AVAILABLE THAT
WE CAN USE AS NIH TO HELP
FACILITATE INVESTIGATORS
IMPLEMENTING THESE PLANS, ONE
ARE USING NO COST EXTENSION SO
WHERE THE INDIVIDUAL PROPOSAL
PERIOD MAY BE FOUR OR FIVE YEARS
THERE IS THAT POSSIBILITY FOR AT
THE END OF THE PROJECT. TO
REQUEST EITHER A FIRST, A NO
COST EXTENSION IS GENERALLY
AUTOMATIC. THERE'S A MANUAL
PROCESS BUT WE CAN DO A SECOND
NO COST EXTENSION, AND WE EVEN
HAD CASE HAVE HAD CASES WHERE
THE INVESTIGATORS SUCCESSFULLY
CAN PROPOSE A THIRD, NO COST
EXTENSION AT THE END WITH EACH
ONE OF THOSE BEING UP TO A YEAR
IN DURATION, THAT DOES PROVIDE
THE ABILITY TO EXTEND THE
OVERALL ACTIVE DIRECTORY PERIOD,
UP TO 3 YEARS, TO THE END OF THE
PROJECT.
AND BENEFIT OF BEING ABLE TO
EXTEND THE GRANT PERIOD KNOWING
THE CARRY OVER FUNDS FOR
EXPLOITATION OR UNEXPECTED
PATIENT COSTS CAN BE CARRIED
OVER TO WHEN NEEDED.
SO FOR EXAMPLE IF AN
INVESTIGATOR IN YEAR ONE OF THE
PROJECT IS PROPOSING HAS
BUDGETED IN HE POSSIBILITY FOR
THE PATIENT CHOOSING TO BE
EXPLANTED AT THE END OF THAT ONE
YEAR STUDY PERIOD IN THE PATIENT
FOR WHATEVER REASON DECIDES NOT
TO NECESSARILY BE EXPLANTED AT
THAT TIME, THOSE FUNDS CAN THEN
BE CARRIED OVER INTO FUTURE
YEARS FOR WHEN OR IF NEEDED.
WITH THAT ONGOING PATIENT AND
BEING ABLE TO APPLY THOSE FUNDS
TO SO THAT THE COSTS ARE NOT
NECESSARILY INCURRED BY THE
SUBJECT OR THE HOSPITAL.
SIMILARITY FOR UNEXPECTED EVENTS
IN THE PROJECT, WE CAN CONSIDER
ADMINISTRATIVE SUPPLEMENTS FOR
THOSE UNEXPECTED PATIENT CARE
EXPENSES AND POTENTIAL
ADDITIONAL FOLLOW-UP IF NEEDED
IN SOME OF THE PROJECTS THAT ARE
GOING WELL.
AND THEN THE OTHER OPTION THAT
I THINK HAS BEEN MENTIONEDED
EARLIER IS INVESTIGATORS
APPLYING FOR NEW GRANT
APPLICATIONS FOR LONG TERM
FOLLOW ON STUDIES, OR IF THERE'S
A NEW TECHNOLOGY ITERATION THAT
THEY'RE LOOKING AT PURSUING,
THEN THEY CAN APPLY FOR FUNDING
FOR THAT APPLICATION AND
DEPENDING UPON HOW THEY LINE UP
THEIR INCLUSION ENROLLMENT FOR
THE STUDY CAN RECRUIT PATIENTS
THAT MAY HAVE HAD THE PRIOR
VERSION OF THE TECHNOLOGY TO
ALLOW THEM TO UPGRADE TO THE
NEWEST VERSION THAT IS THEN
BEING SUPPORTED.
NEXT SLIDE.
SO, YOU KNOW, THERE IS A FEW OF
THE PROGRAMMATIC CHALLENGES TO
HIGHLIGHT THAT WE RUN INTO
ACROSS SEVERAL OF THESE VARIOUS
DIFFERENT AWARDS, AND MOST OF
THESE, YOU HAVE NOTED HAVE BEEN
BROUGHT UP OVER THE COURSE OF
TODAY.
THE FIRST, YOU KNOW, WHEN
LOOKING AT NIH, WE HAVE CONTROL
OF RELEASE OF FUNDS DURING
ACTIVE AWARD PERIODS.
ONCE THAT AWARD IS NO LONGER
ACTIVE WE NO LONGER HAVE THE
AUTHORITY OR ABILITY TO GIVE
ADDITIONAL FUNDS OR COVER
ACTIVITIES THAT ARE NOT
RELATED -- OR NOT WITHIN THAT
PROJECT PERIOD.
SO HENCE THE NOTE ABOUT USING
THE NO-COST EXTENSIONS TO CARRY
OVER SOME OF THOSE ADDITIONAL
FUNDS.
FOR ALMOST ALL OF THESE PROJECTS
ARE MILESTONE DRIVEN COOPERATIVE
AGREEMENTS WITH GO, NO-GO
DECISION POINTS THAT MAY BE
BASED ON EITHER SAFETY OUTCOMES
FOR THE STUDY, OR PRELIMINARY
EFFICACY DATA TO SHOW POTENTIAL
IMPROVEMENTS, IN THE STUDIES SO
FOR PROJECTS THAT FAIL TO MEET
THE MILESTONES, THE FUNDS FOR
THE PROJECT MAY BE REDUCED OR
DISCONTINUED, WHICH CAN ADD SOME
ADDITIONAL COMPLEXITY WHEN
LOOKING AT SOME OF THE POST
TRIAL OBLIGATIONS. THE VARIETY
OF OPTIONS THAT CAN HAPPEN WITH
THE INDUSTRY PARTNERS IN THESE
WHETHER THE COMPANY FILES FOR
BANKRUPTCY GOES COMPLETELY OUT
OF BUSINESS AS ACQUIRED OR
DECIDES TO NO LONGER PRODUCE THE
VERSION OF THE TECHNOLOGY
PRODUCED IN THE STUDY.
EACH ONE OF THOSE CAN CAUSE
INDIVIDUAL CHALLENGES AND
DIFFERENT SITUATIONS FOR THE
VARIOUS DIFFERENT PROJECTS.
FOURTH ON THE LIST THERE IS WHEN
THE PRINCIPAL INVESTIGATOR
CHANGES INSTITUTIONS OR DEPARTS
THE ACADEMIA ALL TOGETHER.
AND NOTED EARLIER TODAY, A LOT
OF THE TIES THE PATIENTS HAVE TO
THE STUDIES TO GO BACK TO THE
PHYSICIAN THAT IS RUNNING THE
CLINICAL TRIAL AND IF THEY'RE NO
LONGER ACCESSIBLE FOR THE STUDY,
THOSE CAN HAVE VARIOUS IMPACTS,
AND EACH ONE OF THE SITUATIONS
THAT COULD STAY WITH NEW PI AT
SAME INSTITUTION OR SOME CASES
THE AWARD CHANGES INSTITUTIONS
WHEN THE THE PI MOVES, WHICH CAN
DEFINITELY AFFECT THE PROPOSED
LONG TERM CARE PLANS, AND
SIMILARLY, WHILE THERE ARE
LETTERS OF SUPPORT IN THE
PROJECTS OR PLANS FROM A
UNIVERSITY PROVIDED SUPPORT, IF
THE UNIVERSITY LEADERSHIP
CHANGES, WHETHER THAT'S AT THE
DEPARTMENT LEVEL, ON UP THROUGH
TRANSFER, AND UNIVERSITY WIDE
POLICIES CAN THAT CAN CHANGE.
EVEN NOTED IN THE CASE OF OREGON
YOU CAN HAVE LAWS THAT CAN
CHANGE IN THE STATE THAT CAN
INFLUENCE SOME OF THOSE PLANS.
AND LAST POINT, IS THE PATIENT
DOES HAVE THE CHOICE TO EXPLANT
AT THE CONCLUSION OF THE STUDY.
NIH HAS NO INTERACTIONS WITH THE
PATIENT AND IS NOT PRIVY TO
WHICH INFORMATION IS SHARED WITH
THEM AS TO ENSURE THAT SOME OF
THESE OPTIONS AND THE FINANCIAL
IMPLICATIONS WITH THEM ARE FULLY
CONVEYED AS IT RELATES TO THE
MANAGEMENT OF THE GRANT AND
WHETHER OR NOT THOSE FUNDS COULD
POTENTIALLY BE AVAILABLE.
SO THAT'S THE END OF MY
PRESENTATION, HAPPY TO TAKE ANY
QUESTIONS, AND GO BACK TO
RHONDA, OR TURNOVER TO THE Q&A
PORTION OF THE PANEL.
>> YEAH, LET'S TAKE A QUESTION.
CHRISTINE, GO AHEAD, PLEASE.
>> THANK YOU, NICK.
I JUST WANTED TO ASK ONE
QUESTION ABOUT, YOU MENTIONED
THERE WAS AN OPTION OF
PURCHASING INSURANCE, BUT THAT
HAS HAPPENED IN OTHER COUNTRIES
AND NOT UNITED STATES.
ANY QUESTION ARE IS THAT WITH
NIH GRANT FUND, AND NUMBER ONE,
AND WHY NOT UNITED STATES, IS
THAT REGULATORY REASON OR JUST
HASN'T HAPPENED.
>> SO, I THINK IN TERMS OF THE
POLICIES AS TO WHETHER OR NOT
THAT'S ALLOWED WITH GRANT FUNDS,
IT SORT OF DEPENDS ON THE NATURE
OF HOW IT'S DESCRIBED IN THE
POLICY, AND GRANT APPLICATION,
WHETHER IT'S ALLOWABLE OR NOT:
IN THEORY, COULD BE -- AS LONG
AS JUSTIFIED TO THE RESEARCH
TOOLS OF THE STUDIES AND
ACCOMPLISHING THOSE.
THE CHALLENGE THAT INVESTIGATORS
HAVE FACED, AND IN PARTICULARLY
NOTED EARLIER.
YOU ARE SORT OF LOOKING AT
TRYING TO HAVE AN INSURANCE
POLICY IN AN AREA WHERE YOU ARE
DEALING WITH SMALL NUMBER OF
PATIENTS, THAT -- THE WAY MOST
INSURANCE WORKS IS MATTERS OF
SCALE, AND LOOKING TO TRY AND
SUPPORT A POLICY OF TRIAL OF 5,
10, 15 SUBJECTS, THE
INVESTIGATORS THAT HAVE TRIED TO
REACH OUT TO INSURERS, HAVE HAD
LIMITED SUCCESS IN GETTING THEM
TO SUPPORT AN ACTIVITY LIKE THAT
FROM A FINANCIAL STANDPOINT.
FROM COST BENEFIT RATIO.
DOESN'T MEAN THERE ISN'T AN
OPTION OUT THERE, AND OTHER
PROVIDERS THAT MAY BE INTERESTED
IN OPERATING IN THE SPACE.
>> THANK YOU.
>> THANKS, NICK.
LET'S -- RHONDA SHALL WE COME
BACK TO YOU.
I THINK YOU MAY HAVE HAD A
LITTLE BIT TO SAY BEFORE WE LOST
YOU AND LET YOU FINISH UP, AND
MOVE ON TO THE PANEL -- OR THE
DISCUSSION PART.
>> ALL RIGHT, LET ME ASK "CAN
YOU HEAR ME?
"
>> YES.
>> OKAY.
I DON'T... I REALLY APOLOGIZE
FOR THESE TERRIBLE TECHNOLOGY
PROBLEMS.
I DON'T KNOW WHERE I LEFT OFF.
BUT I HAVE BEEN ABLE TO HEAR THE
LAST PRESENTATION.
I THINK THE ONLY THING THAT I
WOULD WANT TO ADD TO THIS, IS
THE CONSIDERATION OF THE THINGS
THAT IN TALKING WITH COLLEAGUES
THAT SEEM TO BE DELIMITED AND
NEED TO BE ADDRESSED.
ONE OF THE MAJOR ONES WAS GOING
FROM WHAT I CALL INDIVIDUALLY
PROVEN TO POPULATION PROVEN.
AND THIS IS WHERE, I THINK THIS
QUESTION HAS COME UP AROUND
PROVID
PROVIDERS RESPONDING TO PATIENTS
WHO HAVE NEEDS THAT ARE NOT
BEING MET BY TECHNOLOGIES
CURRENT TECHNOLOGIES THAT ARE
OUT THERE.
AND BEGIN TO USE CERTAIN TYPES
OF TREATMENTS OFF LABEL AND IN
SOME CASES HAVE SEEN IMPROVEMENT
IN THOSE INDIVIDUALS WERE OTHER
GENERALS TREATMENTS HAVE NOT
BEEN SUCCESSFUL. THE ROAD FOR
THAT PARTICULAR JOURNEY TO GET
TO THE POINT OF BEING INSURANCE
COVERED IS VERY DIFFICULT.
BECAUSE OF THE FACT THAT
PROVIDERS GENERALLY DO NOT HAVE
ACCESS TO ENOUGH PATIENTS THAT
THEY ARE ABLE TO APPLY THIS
TECHNOLOGY UP LABEL, AND
THEREFORE, IT WILL NEVER REACH
THE LEVEL OF THRESHOLD FOR BEING
PROVEN, EVEN FOR OBSERVATION
SETTING THAT GENERALLY REQUIRES
AT LEAST 1000S OF PATIENTS TO BE
INVOLVED IN THAT.
I THINK THAT'S A VERY IMPORTANT
PIECE TO TAKE A LOOK AT.
MANY TIMES A PLAN WILL REVIEW AN
INDIVIDUAL WHO HAS HAD
DIFFICULTY TREATMENT RESISTANT
WHETHER IT'S DEPRESSION OR OTHER
TYPES OF CONDITIONS, WITH
CURRENT TREATMENTS AND WE'LL
REVIEW IT ON AN INDIVIDUAL
BASIS.
LOOKING FOR ALL THE DATA OF ALL
THE TREATMENTS RENDERED AND
MAKING SURE THE TREATMENTS WERE
RENDERED AT THE MOST EFFICACIOUS
DOSAGES AND LENGTH OF TIME AND
CAN MAKE AN INDIVIDUAL DECISION.
THOSE ARE DIFFICULT ISSUES
BECAUSE IT REQUIRES A LOT OF
DOCUMENTATION AND IT IS
DEPENDENT UPON THE CLINICAL
INTERPRETATION BY THE REVIEWER
IN THAT CASE.
SO TO ME IT SEEMS AS THOUGH THIS
IS ONE OF THE AREAS WHERE THERE
IS A NEED FOR POLICY, OR THERE
IS A NEED FOR PROCESS.
THE OTHER THING I MENTIONED WAS,
INCLUDING IN CLINICAL RESEARCH,
WHAT I CALL COMPARATIVE
RESEARCH, WHERE YOU ARE
COMPARING A NEW TECHNOLOGY, NOT
TO PLACEBO BUT OTHER EXISTING
TECHNOLOGIES, PARTICULARLY
THAT'S IMPORTANT BECAUSE ONE
WANTS TO UNDERSTAND IF A NEW
TECHNOLOGY IS COMING ON BOARD,
AND ITS EFFICACIOUS HOW WELL DID
IT WORK AGAINST WHAT IS ALREADY
OUT THERE.
AND TWO, TAKING A LOOK AT WHAT
MIGHT BE THE COST DIFFERENTIAL.
AND THIS IS HOW PAYERS WILL
THINK ABOUT THIS IN THAT REGARD.
AND I'LL STOP WITH THAT.
AND AGAIN, APOLOGIZE AGAIN FOR
THE TERRIBLE TECHNOLOGY ISSUES.
>> NO, NO, PROBLEM.
WE ARE GLAD WE COULD GET THAT
IN.
IT'S ALL VERY IMPORTANT
INFORMATION TO FOLD INTO THE
DISCUSSION.
ANY DIRECT QUESTIONS FOR DR.
ROBINSON BEALE?
SASKIA, YEAH,
>> I'LL GO AGAIN.
THANK YOU SO MUCH.
IN SOME OF THE PREMEETINGS WE
LEARNED A COUPLE OF INSTANCES
MAY GENERALLY BE COVERED BY
PRIVATE INSURANCE.
AND I JUST WANTED TO CHECK WITH
YOU IF YOU HAVE THE SAME
IMPRESSION, WE LEARNED THAT
THINGS INCLUDING EMERGENCY
MEDICAL CARE, RELATED TO A
DEVICE.
EXPLOITATION BECAUSE OF MEDICAL
INDICATION.
AND ROUTINE SERVICES THAT A
PATIENT WOULD HAVE REQUIRED
REGARDLESS OF THE EXPERIMENTAL
STATUS OF THE DEVICE.
THOSE ALL WOULD BE COVERED
GENERALLY SPEAKING BY PRIVATE
HEALTH INSURANCE INDEPENDENT OF
WHETHER A DEVICE HAS BEEN
COMMERCIALLY APPROVED FOR
CERTAINLY INDICATION.
IS THAT ABOUT RIGHT?
>> YES, THAT'S CORRECT.
I'M SORRY IN THE PART THAT GOT
BLANKED OUT I DID GO OVER THAT.
I DIDN'T KNOW WHETHER THAT WAS
HEARD.
ABSOLUTELY TRUE, MOST INSURERS I
TALKED TO INCLUDED THAT IN THEIR
PARTICULARLY COVERAGE.
IT KIND OF AGAIN POINTS TO AN
OPPORTUNITY AS NIH APPROACHES,
SENDING OUT RFIS RFPS OR
PARTICULAR TECHNOLOGY AS
PLANNING WORK WITH INSURERS SO
WE UNDERSTAND WHERE THERE MAY BE
GAPS.
PARTICULARLY IF ONE IS ABLE TO
PUT TOGETHER WHAT'S CALLED A
JOURNEY MAP OF WHAT A PATIENT'S
GOING TO BE EXPERIENCING IF THEY
TAKE PART IN THAT PARTICULAR
TECHNOLOGY. AND I THINK KNOWING
UPFRONT WHAT THE ISSUES ARE WILL
HELP INSURERS WORK WITH CLINICAL
TRIAL RESEARCHERS TO HELP CREATE
SOME CONSISTENCY AS MUCH AS
POSSIBLE.
GIVEN THE CURRENT RULES AND
POLICIES IN PLACE.
AND THEN, IDENTIFY WHERE THERE
IS NEED FOR NEW THINKING, AND
NEW PROBLEM-SOLVING.
>> GREAT, THANK YOU.
DID YOU HAVE A COMMENT TO THAT?
>> NO, ACTUALLY TO ALL OF THIS.
SO, WHAT I'M HEARING, IS THAT WE
HAVE THAT FDA PROCESS FOR
DECIDING WHETHER SOMETHING IS
SAFE AND EFFECTIVE.
CMS HAS THEIR PERSPECTIVE, THE
INSURERS HAVE THEIR PERSPECTIVE.
AND OF COURSE THE CONSUMER
COMMUNITY HAS THEIR PERSPECTIVE.
OFTEN HAS TO DO NOT WITH THE
PRIMARY END POINT OF STUDIES BUT
QUALITY OF LIFE.
AND THIS IS VERY INEFFICIENT
PROCESS.
SO IF YOU CONDUCT A TRIAL THAT
IS SATISFACTORY TO FDA IT MAY
NOT BE SATISFACTORY TO CMS AND
TO PRIVATE INSURERS.
AND THIS HAS TO BE RESOLVED.
AGAIN, GO BACK TO MY PERSONAL
EXPERIENCE, BECAUSE IT WAS VERY
POSITIVE.
SO WITH THIS DEVICE, WE ENGAGED
WITH CMS, AND MAJOR INSURERS,
AND BLUE CROSS, BLUE CHEELD TO
HAVE A CONVERSATION ABOUT WHAT
WOULD BE GOOD EVIDENCE.
WHAT WOULD BE SATISFACTORY, AND
DESIGNED A TRIAL TO BE ABLE TO
DO THAT.
AND THAT WORKED VERY WELL.
AND I THINK THAT ESPECIALLY FOR
THOSE WHO ARE DEVELOPING VERY
NOVEL OR INNOVATIVE DEVICES,
LIKE THE ONES THAT NICK IS
FUELING, THESE ARE INDIVIDUALS
WHO MAY NOT BE AS KNOWLEDGEABLE
ABOUT HOW THAT KIND OF ACTIVITY
HAS TO TAKE PLACE EARLIER.
THEY JUST DON'T HAVE THE
EXPERIENCE AND DON'T HAVE
REGULATORY, AND QUALITY.
AND SO MAYBE AS PART OF THE
CLINICAL TRIAL ENTERPRISE, THERE
COULD BE SOME TYPE OF EDUCATION
TRAINING OR ENCOURAGEMENT AS
THESE TRIALS ARE DESIGNED TO
CONSIDER WHAT NEEDS TO BE ON THE
OTHER END.
THE OTHER END IS PROBABLY NOT
FDA APPROVAL.
BUT MAYBE THE KIND OF DATA, THAT
WOULD BE ACCEPTED WORTHY OF
GOING ON A FEASIBILITY STUDY OR
PIFRTAL STUDY.
I FEEL IF EVERYBODY IS MORE
INVESTED ON WHAT IS HAPPENING UP
FRONT, WILL BE EASIER TO BE
INVESTED AFTER THE STUDY IS
DONE.
>> THAT'S A GREAT POINT.
I THINK FOR NOW, IN THE
DISCUSSION PORTION OF THIS,
WHICH IS EXACTLY WHERE WE SHOULD
BE AT THIS POINT, LET'S -- MARTY
IF I MAY, SAY A FEW FRAMING
COMMENTS AND THEN PICK UP RIGHT
WHERE YOU HAVE US.
REMEMBER THIS PANEL -- THE FIRST
PANEL WAS ABOUT ISSUES THAT WE
NEED TO FIX.
THE PANEL IS ABOUT WHAT IS
ALREADY BEING COVERED VERSUS
NOT.
AND WE OBVIOUSLY HEARD A LOT OF
PERSPECTIVES ON THIS.
AND CAN ASK TO FRAME THE WHOLE
DISCUSSION WITH NEUROLOGICAL
FOUNDATION, AND CONSIDER RESPECT
TO PERSONS AND ALL THE THINGS
YOU BILLED ALL THESE PRACTICAL
APPROACHES.
AND THE SPIRIT OF BEING
PRACTICAL, LET'S DO SO.
AND I WAS GOING TO SUGGEST
MARTY, THAT WE START WITH THE
EXAMPLE YOU GAVE.
WITH YOUR LGS TRIAL, WHERE IT'S
DEMOCRATIZED SORT OF DIVIDE AND
CONQUER APPROACH, WHERE THE
DEVICE MANUFACTURER THE PAYER,
WHETHER IT'S GOVERNMENT OR A
PRIVATE INSURANCE COMPANY,
HEALTH CARE SYSTEM,
INVESTIGATOR, THE PATIENT,
FEDERAL SPONSOR, OF THE GRANT,
YOU KNOW ALL HAVE PIECE OF
BURDEN, AND PRICE TO PAY.
AND I WONDER IF WE CAN START
WITH THAT AS A RUBRIC THAT WE
CAN EITHER CONTINUE TO ACCEPT
AND WIDDLE, OR, YOU KNOW, DECIDE
ON -- THIS DOESN'T WORK AT ALL.
I WOULD LOVE TO HEAR FROM THE
VARIOUS REPRESENTATIVES IN
INVOLVED WHETHER THIS IS A
RUBRIC THAT COULD WORK.
YOU KNOW, THE COSTS ARE LARGE,
AND TRIALS ARE EXPENSIVE.
DOES THE DIVIDE AND CONQUER
APPROACH MAKE SENSE FROM THE
PERSPECTIVE OF THE VARIOUS
INDIVIDUALS AND STAKEHOLDERS
REPRESENTED HERE. .
>> LET ME JUST ADD, THE COSTS
COULD BE NORMAL.
BUT IF EVERYTHING WENT WRONG,
AND THE STUFF THAT WE TALKED
ABOUT COULD HAPPEN.
HIGHLY UNLIKELY, YOU CAN'T
ANTICIPATE.
WHAT THEN.
AND SO TALKING TO COMPANIES TO
CONSIDER OUT OF THE BOX
INSURANCE POLICIES, AND I HAD
ONE COMMENT FROM CARL THAT IT'S
SO HARD TO CALCULATE.
IT'S PROBABLY NOT GOING TO BE
MUCH OF ANYTHING.
BUT, IT COULD BE SOMETHING VERY
LARGE.
AND BECAUSE THESE ARE TRIALS,
THERE IS SO MUCH UNKNOWN.
AND SO IT'S VERY DIFFICULT TO
HAVE ANY KIND OF COMPETENT
EQUATION OF RISK.
>> SO LET ME BE SPECIFIC TO GET
STARTED.
CARL, THANK YOU FOR THE
PRESENTATION, I THINK THE
EXAMPLE OF CED WAS A VERY
ILLUMINATING ONE.
ANOTHER ONE THAT I WONDER WOULD
BE RELATED TO THE SITUATIONS WE
HAVE HERE OFTENTIMES IS THIS:
WHICH IS THAT, THERE IS A TRIAL
FUNDED, AND FEDERALLY FUNDED.
AND IT'S A SIGNIFICANT RISK
DEVICE, AND HAS AN IDE APPROVAL
FROM THE FDA.
WHAT ARE THE MECHANISMS BY
WHICH CMS WOULD ASSIST WITH
COVERING THE COSTS OF, LET'S SAY
DEVICE REPLACEMENT YEARS LATER
AFTER THE PATIENT WAS DONE WITH
THE TRIAL, SUPPOSE THE
MANUFACTURER IS WILLING TO
DONATE THE DEVICE, AND THE P
PATIENT DOESN'T HAVE PRIVATE
INSURANCE THAT WILL COVER THE
COST OF THE JUST THE SURGICAL
CARE, THE HOSPITAL CARE. WHAT
DOES CMS ABLE TO DO IN THAT
INSTANCE. .
>> AGAIN, IT'S A LOT OF IT
DEPENDS ON YOUR SITUATION,
BECAUSE EVERYBODY'S I WOULD
ACTUALLY WANT TO KIND OF MAYBE
BACKTRACK A LITTLE BIT AND I
KNOW ONE OF THE SLIDES THAT I
THINK THE LAST PRESENTER PUT UP
TO AND YOU JUST DISCUSSED IT I
WANT TO SAY THAT IT HAPPENS TO
BE MY GROUP BY SELF DIRECTED
COLLECTION WERE INVOLVED WORKING
WITH THE FDA ON THESE IDE
SWITCHES, WHICH MEANS DEVICE FOR
THE EARLIEST STAGE OF APPROVAL
THROUGH THE FDA. AND SO, CMS
HELPS TO MAKES COMMENTS ON THE
CLINICAL TRIALS GOING ON THERE,
EARLY FEASIBILITY STUDIES AND
MAKE SURE THE EVIDENCE COLLECTED
IN THE EARLY FEASIBILITY STUDY
HELPS PROVIDE EVIDENCE THAT CMS
COULD THEN REVIEW TO DETERMINE
WHETHER OR NOT SHOULD CONTINUE
TO PROVIDE COVERAGE.
EVEN AFTER PIVOT TRIAL.
SO WE WERE INVOLVED EARLY ON.
NOW, IF THAT EVIDENCE LEADS TO
NATIONAL COVERAGE DETERMINATION
OF THE SPONSOR REQUESTS NATIONAL
COVERAGE DETERMINATION, AFTER
THE IDE, ALSO COMES INTO THE
PLAY.
BEING THAT.
IF THERE IS ENOUGH EVIDENCE THAT
DEVICE SHOULD BE COVERED.
THE DEVICE AND EVIDENCE FOR MOB
CLINICAL TRIALS.
AND OBSERVATIONAL STUDIES THAT
HAVE OCCURRED, AGENCY WILL COVER
DEVICE AND ROUTINE SERVICES FROM
THAT POINT FORWARD.
AND ONCE THE NCD HAS GONE
THROUGH THE PROCESS, AND
APPROVED, AGENTS THEN SHOULD
COVER IT.
SO IT'S A LONG PROCESS.
>> WHAT ABOUT EARLIER IN
PROCESS, THERE IS QUESTION BY
APHONNOWS ATTENDEE.
2:47 SEN RAL TIME ANYWAY IF
PEOPLE WANT TO CHECK THE Q&A.
AND I'LL JUST PARAPHRASE AN IDE
STUDY OFTENTIMES, BY DEFINITION,
EARLY IN THE PROCESS.
AND SO NOT GOING TO BE EVIDENCE
OF EFFICACY OF THAT DEVICE.
AND MARTY HAS YOU SAID, WE
SHOULDN'T ENTER INTO THESE
THINGS PERHAPS WITHOUT HAVING
THOUGHT ABOUT THEM AHEAD OF
TIME. DISCIPLINARY APPROACH,
RIGHT. SO WE SHOULDN'T START
TRIALS WITHOUT HAVING SOME SENSE
OF OF HOW THEY'RE GONNA GO WITH
AN IDE WE DON'T HAVE WE'RE NOT
GOING TO HAVE THE EVIDENCE
SUGGESTING THE SHOWING EFFICACY
AND SO WE WOULD, WE WOULD HAVE
TO ENTER THAT WITHOUT THE
EVIDENCE, BUT THE PATIENT
PARTICIPANT WILL HAVE TO KNOW
WHAT IS GOING TO HAPPEN WITH
THEM AFTER THEY LEAVE THE TRIAL
WAS REALLY BEFORE THERE'S ENOUGH
EVIDENCE FOR AN NCD ETC. WHAT
ABOUT THAT SITUATION.
SO -- THAT'S A WHOLE ANOTHER
PRESENTATION, I DID NOT -- I
HAVE THE SLIDE, BUT NOT GOING TO
PUT YOU THROUGH THAT.
DURING THAT ID PROCESS.
WE WORK CLOSELY WITH FDA.
THE POINT IS WE ACTUALLY... WE
ARE WILLING TO ACCEPT A LOT
DETAIL, BUT SEEN SAMPLE SIZE OF
5 THAT SHOWS EFFICACY, AND NO
HARMS, AND BENEFITS OUTWAY THE
HARMS, AND WILL ACTUALLY PROVIDE
COVERAGE FOR THE DEVICE AND THE
RELATED SERVICES, EVEN AT THAT
EARLY STAGE SO DURING THE EARLY
FEASIBILITY STUDY, I MEAN, THE
FDA CLEARED IT. AND, YOU KNOW,
BELIEVES THAT THE, YOU KNOW,
AGAIN, THE BENEFITS. THE
BENEFITS OUTWEIGH THE HARMS ARE
WILLING TO PROVIDE COVERAGE FOR
THE DEVICE AND SERVICES EVEN
DURING THE EARLY FEASIBILITY
STUDY DURING THE ID PROCESS.
SO, AGAIN IT DEPENDS ON THE
EVIDENCE, BUT WE UNDERSTAND THAT
VERY EARLY IN THE FDA APPROVAL
PROCESS, THERE MIGHT BE VERY
LITTLE EVIDENCE BUT WE DO --
DON'T WANT TO SAY REQUIRE -- BUT
WE'RE INTERESTED IN LOOKING AT
INTERNATIONAL STUDIES OF SAMPLE
SIZE OF 5.
AND PERHAPS THE EVIDENCE THERE
IS PROMISING, AND WILLING TO
TAKE THAT UP AS AS EVIDENCE OF
POTENTIAL EFFICACY OF THE DEVICE
AT THAT EARLY STAGE .
>> MARTY IS THAT A FRESH RAISED
HAND.
>> NO, PROBABLY AN OLD ONE, BUT
I WAS GOING TO RAISE MY HAND.
SO THAT'S AN EFFICIENT HAND.
I'LL TAKE MY HAND DOWN.
DR. LI, I WOULD LIKE TO SAY,
POLICY THAT CMS HAS FOR COVERING
INVESTIGATIONAL TRIALS UNDER
THESE CIRCUMSTANCES THAT YOU
JUST OULTED, AND NOW MEDICAID, I
UNDERSTAND THAT CONGRESS ENABLED
THAT A COUPLE MONTHS AGO IS ONE
OF THE REASONS WHY THINGS ARE
HANG IN UNITED STATES, AND IT'S
ALSO PROVIDINGS GUARANTEESES
THAT STUDIES ARE WELL THOUGHT
OUT AND SCIENTIFICALLY BALANCED
AND THAT THE ENDPOINTS ARE
MEANINGFUL JUSTIFIABLE. AND SO
BUT WE GET IN THE SAME PLACE AS
IT WANTS TO STUDIES DONE WHAT
HAPPENS TO THE PATIENT. AND THIS
IS, YOU KNOW I THINK UPFRONT,
WE'RE ALL ACTUALLY DOING REALLY
WELL. WE HAVE NIH THAT'S DOING
EXTRAORDINARY WORK THROUGH, FROM
MY PERSPECTIVE THE BRAIN
INITIATIVE AND THE PUBLIC
PRIVATE PARTNERSHIP WHICH IS
REALLY QUITE INNOVATIVE, AND
THAT'S REALLY EFFECTIVE. WE HAVE
FDA WHO HAS BECOME REALLY QUITE
ENGAGED AND FLEXIBLE, AND TRYING
TO PROMOTE AND ADDRESS YOU WILL
A THE CHECKS, AND TECHNOLOGY,
WHICH HAPPENS SO QUICKLY THAT
IT'S HARD AND REGULATORY
ENVIRONMENT TO KEEP UP WITH THEM
ALL. NOW WE HAVE THIS BUT THEN
NONE OF US KNOW WHAT TO DO IN
THE PATIENT FINISHES.
IF THE STUDY ISN'T SUCCESSFUL,
IF THE DEVICE DOESN'T RECEIVE
APPROVAL AND CMS DECIDES THAT
THE EVIDENCE IS NOT WHAT THEY
WANT OR THE PRIVATE INSURER SO
THIS IS WHERE WE HAVE THE ISSUE
AND RHONDA WE NEED AN INSURANCE
POLICY, WE NEED AN INSURANCE
POLICY THAT EVERYBODY PAYS INTO.
BECAUSE AGAIN YOU CAN'T ESTIMATE
THE RISK.
PROBABLY EVERY R -- THE RISKS AE
VERY LOW.
AND SO THESE ARE MY OPINIONS.
>> LET ME FOLLOW-UP ON THAT.
AND THEN SHIRLEY COME TO YOU IN
A SECOND.
AND SO LOOK AT THE QUESTION FROM
THE Q&A, AND ALSO RHONDA FOR
YOU, AND PERSPECTIVE OF PAYER
AND SYSTEM IS PAYING.
HOW QUALITY OF LIFE,
INCORPORATED INTO THESE
DECISIONS AS TERM OF CAN
COVERAGE.
CAN YOU ANSWER THAT QUESTION
ALONG WITH MARTY'S?
>> YES.
VERY GOOD QUESTION.
SO QUALITY OF LIFE IS ONE OF THE
FACTORS THAT ONE LOOKS AT WHEN
THEY ARE JUDGING THE TECHNOLOGY
IN ITSELF.
IT KIND OF GOES BACK TO LOOKING
AT THE CONDITION ITSELF.
AND WHAT IT DOES TO THE QUALITY
OF THAT INDIVIDUAL'S LIFE, AND
THE LIKELIHOOD OF DISABILITY,
LIFELONG DISABILITY.
THE PROBLEM WITH THAT, IS MANY
OF THE INSURERS DON'T
NECESSARILY HAVE A DESIGNATED
POLICY, BECAUSE DISABILITY IS A
VERY BROAD SUBJECT.
AND IT'S VERY HARD TO PUT
QUALIFIERS HOW DISABLED SOMEONE
NEEDS TO BE.
AND WHEN ARE THEY CONSIDERED
DISABLED AND WHAT ARE THE
DEFINIT
DEFINITIONS, AND IN ORDER TO
CREATE A SUBSET OF CONDITIONS
AND LEVELS OF DISABILITY, SO
THAT ONE CAN BECLEAR
OUTLINELINE, WHEN SMIOMETHING I
COVERED BY A DISABILITY AS
OPPOSED TO SOMETHING THAT WAS
VERY CLEAR IF SOMEONE'S GOING TO
DIE IN A YEAR, OKAY? IT'S A
LITTLE EASIER TO MAKE THAT
DISTINCTION. DISABILITY IS VERY
WIDE, WIDELY DEFINED AND THERE'S
SO MANY DIFFERENT LEVELS. SO I
THINK THAT MIGHT BE WHY YOU
DON'T SEE POLICIES SO TO SPEAK,
IN WRITING THAT DEAL WITH
DISABILITY OR QUALITY OF LIFE AS
A DETERMINANT IN DECISION
MAKING, IT IS CONSIDERED NOT
NECESSARILY A DRIVER UNLESS ABLE
TO DEFINE DISABILITY AND ABLE
TO PUT IN SOME TYPE OF
DEFINITION, AND SOME TYPE OF
REGULATION THAT WOULD ALLOW THAT
INVESTIGATION AND CONSIDERATION
IN TERMS OF COVERAGE.
>> THANKS.
SHIRLEY.
>> LET ME LOWER MY HAND NOW.
HAVE A COUPLE OF THOUGHTS.
AS A RESEARCH ADMINISTRATOR IT
IS MY MORAL OBLIGATION, TOLESSEN
ADMINISTRATION AS MUCH AS
POSSIBLE.
BECAUSE IT JUST SEEMS LIKE A --
THAT GETS IN THE WAY OF ANYTHING
HAPPENING.
I HAVE A COUPLE OF THOUGHTS
ABOUT STANDARD OF CARE, STANDARD
OF CARE DOESN'T EVEN WORK, WE
GET DENIALS ALL THE TIME, AND
SEEMS LIKE THE GO-TO MOVE IS
JUST TO DENY.
I HAVE A COUPLE EXAMPLES.
PATIENT HAS BRAIN SURGERY, SKULL
CAP COMES OFF, AND DENIAL TO PUT
IT BACK, IS DENIED BECAUSE IT'S
CONSIDERED CONSTANT METTIC, AND
SO THE PATIENT IS SUPPOSED TO
WANDER AROUND WITH THE BRAIN
HANGING OUT OF THEIR HEAD.
THAT'S EXTREME EXAMPLE.
BUT WE GET THOSE DENIALS AND
STANDARDS OF CARE WITH DEEP
BRAIN STIMULATION, WE STILL
ROUTINELY GET DENIALS FOR DEEP
BRAIN STIMULATION TO TREAT
DYSTONIA, BECAUSE IT WAS
APPROVED IN 2003 AS A
HUMANITARIAN DEVICE THAT WAS 20
YEARS AGO. SO I GUESS MY
QUESTION IS: DO YOU WANT TO FIX
THE STANDARD OF CARE SYSTEM?
OR DO YOU WANT TO TAKE WHAT
HAPPENS IN CLINICAL TRIAL
RESEARCH, AND BE INNOVATIVE.
AND AS MARTHA SAID PUSH THAT TO
PUSH STANDARD OF CARE TO ADAPT
AND BECOME BETTER ALSO.
AND SOMEONE WHO GREW UP IN
SOCIALIZED COUNTRY, I STRUGGLE
WITH THAT YOUR HEALTH CARE
BENEFITS ARE TIED TO ACTUALLY
HAVING A JOB THAT PROVIDES YOU
THAT BENEFIT, WHICH I DON'T SEE
AS A BENEFIT, BUT OTHER THAN
THAT. AND ANOTHER THING ABOUT
THE FDA, I HAVE NOTICED CHANGES,
WHICH I THINK ARE GOOD. HOWEVER
MY STUDY COORDINATOR CAME AND
SAID, YOU HAVE A CD LIKE WHAT DO
YOU NEED TO HAVE A CD FOR. THE
FDA WANTS ME TO SEND THESE
DOCUMENTS ON A CD. I'M LIKE,
YOU'RE ABSOLUTELY KIDDING ME
RIGHT. SO WE HAD TO SPEND TIME
FOR A CD, AND THEN NOT EVEN THEN
WE HAD TO FIND A COMPUTER THAT
HAD A CD DRIVE, AND THEN A CASE
TO PUT IT IN. SO, WE ARE MAKING
PROGRESS BUT WHEN IT COMES TO
THE ADMINISTRATION, WHICH IS
WHAT I WANT TO FIX. THOSE ARE
JUST COMMENTS FOR YOU TO TAKE
AWAY WITH YOU TODAY. .
>> THANK YOU FOR TRYING TO
REDUCE THE ADMINISTRATIVE BURDEN
FOR OUR CUSTOMERS.
YOU HAD YOUR HAND UP EARLIER.
>> IF YOU FOR MAKING US
PARTNERS IN THERE, FROM EVERY
STANDPOINT, AND I HAD A QUESTION
FOR DR. LI, IN THE SENSE THAT,
YOU KNOW, AS CLINICIAN MYSELF
AND RESEARCHER, AND FIND THE CD
MECHANISM FOR VAGUS NERVE
STIMULATION IS REALLY TANGIBLE
OPTION FOR PATIENTS WHO
OTHERWISE, NOT ABLE TO BE ABLE
TO ENCONTROL IN NIH SPONSORED
TRIALS AND FOUR WEEKS OFF LABEL
PROCEDURES, AND THE VAGUS NERVE
STIMULATION APPROVAL BY FDA IN
2005.
LED TO A PERIOD OF, YOU KNOW,
15, 20 YEARS OF DROUGHT UNTIL
THIS NEW MECHANISM.
IS THIS, YOU KNOW, CD WITHOUT AN
FD APPLICATION BEFORE,
FORESEEABLE FOR TRIALS THAT
INVOLVE, FOR EXAMPLE DEEP BRAIN
STIMULATION FOR NEW INDICATIONS,
WHETHER IT'S, YOU KNOW,
DEPRESSION OR OTHER PSYCHIATRIC
INDICATI
INDICATIONS..
>> , YOU KNOW, AGAIN, EACH
SITUATION IT'S NUANCED.
BUT I WOULD SAY IN GENERAL, OUR
MECHANISMS, LIKE THE OTHER
PATHWAY IS THE IDE PROCESS, THAT
REQUIRES THAT INVOLVEMENT,
THAT'S ONE OF OUR MAJOR PATHWAYS
WITH THE CMS COVERAGE.
AND THAT'S THE ID PROCESS,
INVOLVED IN FDA.
AND THE CD PROCESS, CVD PROCESS,
WHEN THE SPONSOR OR MANUFACTURE
COMES TO US INDIVIDUALLY, AND
REQUEST NATIONAL COVERAGE.
I CAN ONLY SPEAK TO SOME OF THE
NTDS I'VE WORKED ON, ALL WE
REQUIRE FDA APPROVAL OF THE
DEVICE FOR THE SERVICE. BEFORE
WE EVEN CONSIDER LOOKING AT THE,
AT THAT QUESTION. ON THE OTHER
HAND, THERE ARE... THERE IS
ANOTHER PATHWAY, WHERE IF THERE
ARE QUESTIONS ABOUT FDA APPROVAL
PROCESS, I MEAN, YOU HAVE
SOMETHING CALLED LOCAL MEDICARE
ADMINISTRATIVE CONTRACTS THAT
LOOK AT LOCAL COVERAGE OF THE
ITEM OR DEVICE.
>>KARL: WE SUCCESSFULLY DONE
THAT FOR OUR PATIENTS WHO
RECEIVE DEEP BRAIN STIMULATION
FOR DEPRESSION AT EMORY, AND
IT'S A UNIQUE ARRANGEMENT WE
HAVE THAT MEDICARE COVERAGE FOR
US, AND VERY THANKFUL FOR THAT.
AND SOMETIMES SEE THE CD
APPROACH AS WAY OF HAVING
MEDICARE FUNDED KLEINICAL TRIAL.
AND, YOU KNOW, BEING TONGUE IN
CHEEK.
>> THAT IS THE PURPOSE.
BUT, TO DEVELOP THE EVIDENCE TO
PROVIDE NATIONAL COVERAGE
DETERMINATION.
SO I MEAN, THAT IS IN FACT THE
REASON FOR THE CDE PROCESS.
EVIDENCE IS STILL SOMEWHAT
EARLY, AND PROMISING, FOR
EXAMPLE, OBSERVATIONAL STUDIES
MAY BE TOO SMALL AND NOT THE
BEST STUDY.
SO THE EXAMPLE OF PROVIDING
STANDARDIZED, AND, YOU KNOW,
THAT EVIDENCE IS SHOWS THAT THE
BENEFITS OUTWEIGH THE RISKS. THE
ITEM RELIES ON THE FACT THAT
THEY WILL PROVIDE A NATIONAL
COVERAGE, RIGHT, BUT THE ISSUE
THAT WE HAVE, AS INVESTIGATORS
MANY TIMES HAS BEEN, YOU KNOW,
IS THAT THE STANDARDS FOR FDA
APPROVAL, GO BEYOND, YOU KNOW
THE DOCUMENTATION OF
SIGNIFICANT LONG TERM DATA
SHOWING EFFICACY, LIKE THE
INITIAL FDA APPROVAL FOR VAGUS
NERVE STIMULATION IN 2005 AT
CLINICAL TRIAL THEY HAVE NOT
BEEN THAT STRONG, BUT THAT THE
FDA APPROVAL, THEN THEY STOPPED
COVERING FOR IT IN CMS, AND TOOK
COMBINED PAPER OF 800 PATIENTS,
YOU KNOW, DETERMINING LONG TERM
DATA, SOMETHING THAT WOULD GO,
CERTAINLY OUTSIDE THE COVERAGE
OF ANY CLINICAL TRIAL THIS FIVE
YEARS, 795 PATIENTS BE
FINANCIALLY VIABLE, AS YOU KNOW,
I ASSUME, BY A SINGLE COMPANY TO
FUND AND SUPPORT. SO ALL THE
STAKEHOLDERS HERE COULD SEE A
LONG TERM PLAN FOR PATIENTS,
ESPECIALLY WHEN -- THE LAST
THING I WANT TO SAY.
THE REASON WHY WE ARE SO
INVESTED IN POSTCLINICAL TRIAL
CARE, IS THAT IN MANY CASES,
I'VE BEEN PRIVILEGED TO TREAT
TWO OF MY PATIENTS THAT ARE HERE
IN THE PANEL TODAY.
THE BENEFITS, ESPECIALLY IN THE
CHRONIC CONDITIONS, MANIFEST
LONG AFTER THE ARBITRARY END
POINT OF A PRIMARY CLINICAL TIME
POINT, 6 MONTHS OR 12 MONTHS,
ESPECIALLY TALKING ABOUT
FUNCTIONALITY.
RECOVERY.
THESE TRIALS TAKE YEARS.
THAT'S WHY IT WAS SO IMPORTANT
ON THAT VAGUS NERVE STIMULATION
STUDY, TO DEMONSTRATE CUMULATIVE
EVIDENCE OVER 5 YEARS, SOMETHING
NOT FEASIBLE IN STANDARD
CLINICAL TRIAL DERIVED FROM DRUG
TRIALS, 12 WEEKS.
YOU KNOW, YOU WILL NEVER
DEMONSTRATE THAT.
SORRY, THAT WAS MY COMMENT.
>> I'LL TRY AND ANSWER THE
QUESTION, AND I'LL START OFF BY
SAYING THAT, YOU KNOW, THE
PURPOSE -- OR WHAT FDA LOOKS AT
AND CMS LOOK AT ARE ACTUALLY
COMPLEMENTARY.
AND FDA LOOKS LIKE, LOOKS AT THE
SAFETY OF THE DEVICE.
CLINICAL VALIDITY, DOES THE
DEVICE TEST ACTUALLY TEST WHAT
IT'S SUPPOSED TO BE TESTING.
AND LOOK AT CLINICAL UTILITY.
AND LOOK AT WHETHER OR NOT, ITEM
HAS LONG TERM IMPROVEMENT, YOU
KNOW, THE OUTCOME, SUCH AS
AGAIN, MORE WE USE MORTALITY,
LIKE GOLD STANDARD, BUT IT CAN
BE OTHER OTHER OUTCOMES LIKE
QUALITY OF LIFE, INCLUDE THAT IN
SOME OF OUR CD STUDIES. AND SO,
BOTH AGENCIES NEED TO LOOK AT
AND DETERMINE WHETHER OR NOT,
YOU KNOW, ITEM MEETS THE
OBJECTIVES OF INDIVIDUAL
AGENCIES. SO AGAIN, IN
GENERALITIES AND THERE'S SOMEONE
IN THE FDA IN THIS WORKSHOP, BUT
LOOK AT SAFETY IN PARTICULAR.
CLINICAL VALIDITY, WE LOOK AT
CLINICAL UTILITY AND SO THAT'S
WHY ... .
>> I'M TRULY SORRY TO INTERRUPT
YOU, BUT DO YOU INCLUDE PATIENTS
--
>> THE NCD IS ARE BASED ON
PUBLISHED EVIDENCE.
>> THAT'S NOT WHAT I'M ASKING.
I'M ASKING IF YOU HAVE A PATIENT
ADVOCACY GROUP, BECAUSE THE
PATIENT S CORE AND CENTRAL TO
THE QUESTION, AND CORE OF WHAT'S
GOING ON.
THE ONLY WAY TO ADDRESS EQUITY
AND DIVERSITY IS TO INCLUDE
PATIENTS IN THE CONVERSATION.
>> SHORT ANSWER, THEY ARE NOT
INVOLVED IN OUR NATIONAL
COVERAGE DETERMINATION PROCESS.
>> TYLER YOU HAVE BEEN VERY
PATIENT.
WOULD LOVE TO HEAR YOUR VOICE,
HAD A GREAT PLEASURE OF HEARING
YOU TALK IN ONE OF THE
NEUROSURGICAL MEETINGS A FEW
WEEKS AGO IN PHILADELPHIA.
>> APPRECIATE THAT.
YEAH, I WAS CHATTING WITH NINA
IN THE CHAT, AND TRYING TO SOAK
IT ALL IN.
I WAS GOING TO ASK A SIMILAR
QUESTION THAT DR. MCCARTNEY
ASKED.
NOT JUST IF PATIENTS ARE
INVOLVED IN THE PROCESS, BUT HOW
CAN WE BE?
I HAVE BEEN LISTENING AND
HEARING A LOT ABOUT AWARENESS
AND RAISING FUNDS AND DATA.
AND IT SEEMS LIKE THE BEST WAY
TO GET IT IS FROM THE HORSES
MOUTH.
I'M IN DR. R P STUDY.
AND LIKE BRANDY MENTIONED
EARLIER, I'M CONTINUING TO GIVE
THEM AS MUCH DATA AS POSSIBLE.
IF IT HELPS WITH TREATMENT
RESISTANT DEPRESSION.
I'M ALL FOR IT.
AND I WAS HONORED AND GRATEFUL
TO BE ABLE TO SPEAK IN
PHILADELPHIA, A FEW WEEKS AGO AT
THE AMERICAN ASSOCIATION OF
NEUROLOGICAL SURGEONS AND... I
GUESS... I DIDN'T REALIZE HOW
MANY HANDS ARE IN THE PIE HERE
TRYING TO FIGURE OUT HOW TO DO
THIS.
IT'S A LITTLE OVERWHELMING, BUT,
AGAIN, I GUESS MY QUESTION IS
FOR -- ANYONE CAN ANSWER IT.
ONE AT A TIME PLEASE.
BUT, HOW CAN A PATIENT LIKE ME
HELP AN AS FAR AS NOT JUST
GIVING YOU GUYS MY PERSPECTIVE,
BUT AGAIN RAISING THAT
AWARENESS, AND BEING ABLE TO
PROVIDE THAT DATA.
LIKE, WHAT CAN I DO?
AS AN INDIVIDUAL TO DO THAT?
>> THAT'S IS A GREAT QUESTION.
>> I'D LIKE TO MODIFY MY ANSWER
AND GIVEN IT A LITTLE MORE
THOUGHT.
SO IN MY GROUP IN PARTICULAR WE
HAVE MEDICARE EVIDENCE
DEVELOPMENT AND COVERAGE
ADVISORY COMMITTEE.
SOMETHING CALLED MEDCAC.
NOT SURE IF PEOPLE ARE FAMILIAR
WITH THAT.
THAT'S WHERE WE ASK FOR EXPERT
ADVICE ON DEVELOPING TECHNOLOGY
OR ADVICE. AND SO WE GET
EXPERTS TOGETHER, AND PATIENT
ADVOCATES AND MANUFACTURERS AND
A
ACACADEMIA IN THE FIELD.
AND CAN BE ANY PARTICULAR TOPIC
AND THEN ALSO INCLUDES GROUPS OR
PATIENTS AS WELL, TO WRITE IN
PERSON, ACCESS TO THE FUTURE
DIRECTION OR, YOU KNOW,
PARTICULAR QUESTION REGARDING
HOW TO ACCESS AN ITEM OR DEVICE.
SO THAT WAY WE DO A PATIENT
INPUT.
ALONG WITH THE MANUFACTURES.
AND SO THAT'S USUALLY WHAT'S THE
STATE OF THE ART OF INNOVATIVE
TECHNOLOGY, SO THROUGH THAT
MECHANISM, DO GET INPUT FROM
PATIENTS.
>> GO AHEAD, NINA.
>> I WAS JUST GOING TO MENTION
THAT ON THE AGENDA.
THE DISCUSSION IS HERE SUPPOSED
TO BEGIN TO START TO WRAP UP
NOW.
IT'S SO RICH, WE DO HAVE JUST
A COUPLE OF EXTRA MINUTES OF
WIGGLE ROOM WITH EVERYBODY'S
PERMISSION TO CONTINUE THIS
DISCUSSION FOR A FEW MORE
MINUTES TO MAKE SURE THAT
EVERYBODY'S VOICES ARE HEARD.
AND YES, TRACY, PLEASE. HI I'M
TRACY SALAZAR, I'M EXECUTIVE
DIRECTOR OF THE LENNOX GASTAUT
SYNDROME FOUNDATION LIKE DR.
MOREAU WAS TALKING ABOUT FOR THE
RNS STUDY VERY SEVERE SHAPE
POPULATION SEES YOUR START IN
CHILDHOOD AND THEY BECOME
DISABLED ADULTS... I'VE HEARD A
LOT ABOUT FRAMING VALUE AROUND
ANY POLICY OR RETURN ON
INVESTMENT OR MONEY MADE OR
HELPING PATIENTS, BUT WHAT I NOT
HEARD IS VALUE FRAMEWORK
CENTERED AROUND PATIENT
OUTCOMES.
ONLY PATIENTS CAN WEIGH IN ON
HOW ABSOLUTELY SHITTY THE
DISEASE THAT HE ARE LIVING WITH.
AND WHETHER VALUE HAS BEEN ADDED
TO SOMETHING.
WE DON'T HAVE TO INVENT THIS,
BECAUSE THE FDA FOR THE LAST 10
YEARS, HAS BEEN MANDATED BY
LEGISLATION TO CREATE A PATIENT
FOCUSED DRUG DEVELOPMENT
FRAMEWORK, WHICH CAN EASILY
MOVED OVER TO THINKING ABOUT
DEVICES, WE AT THE FOUNDATION
DEVELOPMENT MEETING AND WE'VE
COMPLETED THE RISK BENEFIT
RATIO, ABOUT WHAT IT'S LIKE TO
LIVE WITH THE CONDITION. WE'VE
TALKED WAS LIKE TO INTERACT WITH
CURRENTLY AVAILABLE TREATMENTS,
AND THE FDA SET THE STANDARDS
FOR SAFETY, EFFICACY AND TOLL
RABLT.
RIGHT, THINKING ABOUT
MEDICATION, AND THEY SHIFTED
THEIR THINKING, I WAS RECENTLY
IN A MEETING WITH FDA WITH A LOT
OF DRUG MAKER AND IS DWAGS
MAKERS AND THE FDA KEPT SAYING
TO CLINICIANS AND THE
RESEARCHERS IN THE ROOM. WHAT IS
IT THAT PATIENTS WANT AND GO A
STEP FURTHER. IT'S IT'S MORE
SOPHISTICATED THAN JUST
ANECDOTALLY ASKING WHAT TRACY
WANTS FOR HER DAUGHTER.
IT'S ACTUALLY THAT PATIENT
ADVOCACY GROUPS ARE DEVELOPING
OUTCOME MEASURES AND THE WORK
THAT WE'VE BEEN DOING IN THIS
SPACE, YOU KNOW OUR KIDS DON'T
ACHIEVE MILESTONES THEY BARELY
ACHIEVED IN STONE, BUT WE SEE
THEM MAKING PROGRESS, , AND YOU
CAN'T MEASURE THE PROCESS OF
MAKING WITH CURRENTLY AVAILABLE
TOOLS, BECAUSE THE WORK THAT
WE'VE BEEN DOING WITH OTHERS, WE
CAN MEASURE THE INSTANT. WE HAVE
FIGURED THAT OUT SO THE FDA IS
DOING A FOUR PART SERIES ON
PATIENT FOCUSED DRUG DEVELOPMENT
THEY PUBLISHED THE FIRST TWO I
ENCOURAGE YOU GUYS TO READ.
THE SECOND AND THIRD, WE'RE
LOOKING AT THE OUTCOME METRICS.
SO I FEEL LIKE THREE THINGS I
THINK THE FIRST THING IS WE NEED
TO SHIFT OUR VALUE FRAMEWORK.
SO WHAT MATTERS TO PATIENTS,
PATIENTS SHOULD BE ON PATIENTS
ARE VERY SOPHISTICATED, I HAVE A
PHD IN NEUROSCIENCE, I'VE BEEN
AN ADVOCATE FOR ALMOST 30 YE
YEARS,I CAN WEIGH IN ON THIS
STUFF FOR EVERYONE ON THIS CALL
HAS BEEN DEALING WITH THIS AT
THE PATIENT HAS BEEN DEALING
WITH YOU CAN WEIGH IN ON EVERY
ASPECT OF DEVELOPMENT, BE
SPEAKING TO EVERYBODY AT EVERY
LEVEL SHOULD BE SPEAKING TO CMS
AND NIH.
NOTHING ABOUT US WITHOUT US.
FIRST THING I WISH I THINK WE
DID HAVE VALUE FRAMEWORK SHIFT
THE WAY WE'RE THINKING ABOUT
THIS. THE SECOND THING I WOULD
SAY IS IT'S ALREADY DONE AND
EXECUTED WITH THE FDA.
SO WE HAVE IT INVENTED.
I THINK WE COULD THINK ABOUT
THIS, AND I LOOK AT WHAT THEY'VE
DONE. AND I WOULD SAY IS THAT I
THINK WE NEED TO FIGURE OUT WHAT
IMPROVEMENT LOOKS LIKE RIGHT? SO
IT'S ONE THING TO SAY YOU KNOW
WE DO CLINICAL TRIALS, IT'S A
50% REDUCTION IN SEIZURES
DECADES, OR HUNDREDS WEEK. IF
YOU DO A 50% REDUCTION IN
SEIZURES TO GET FDA. DID YOU
REALLY MEET THE PRIMARY AND TO
DEVELOP TREATMENTS AND, YES,
SEIZURES ARE CAN BE VERY DEADLY
IN OUR POPULATION AND SO
REDUCING THEM IT'S GOOD BUT YOU
KNOW WHAT IS THINGS THAT REALLY
MATTER IN ADDITION TO THAT, SO I
THINK WE NEED TO SET-- AND WE
CAN DO THIS.
BECAUSE EVERY LITTLE INDIVIDUAL
DISEASE IS HAVING THEIR OWN
PATIENT FOCUSED DRUG DEVELOPMENT
PLATFORM FOR DEVELOPING THEIR
OWN, WHAT VALUE IS. AND THEN
THAT CAN BE THE COMMON THEME
RATHER THAN SORT OF SHELF
PATIENTS INTO A POLICY THAT
WE'RE GOING TO, BECAUSE ALL OF
US ARE IN THIS TO HELP PATIENTS
REALLY WANT TO HELP, WHAT'S
BROKEN IS THE SYSTEM. AND SO
INSTEAD OF TRYING TO USE THE
CURRENTLY AVAILABLE SYSTEM TO
SORT OF SHOVE IN A SOLUTION.
MAYBE WE CAN THINK ABOUT A
LITTLE BIT, JUST AS THE FDA HAS
BEEN DOING FOR THE PAST 10
YEARS. AND SO JUST SOME THOUGHTS
TO CONSIDER. >> THANK YOU TRACY
FOR SHARING THAT.
I THINK THAT MIGHT BE REALLY
ELOQUENT WAY TO CLOSE THIS
PANEL, AND FIRST DAY OF THE
WORKSHOP.
AND I THINK WE'RE ALL TAKING IN
AND SOAKING IN A LOT OF THE
PERSPECTIVES THAT HAVE BEEN
SHARED.
SO I'M GOING TO CLOSE THIS
AFTERNOON'S PANEL, AND TURN IT
OVER TO TO WRAP UP FOR DAY ONE.
>> ALL RIGHT, THANK YOU,
MODERATORS AND PANELISTS FOR THE
GREAT SECOND PANEL.
IT'S A LOT TO PIECE TOGETHER.
I THINK JUST TO PROVIDE A VERY
HIGH LEVEL SUMMERY, I THINK
DURING THE FIRST PANEL TODAY, WE
HEARD ABOUT MANY NEEDS THAT
PARTICIPANTS MAY HAVE AFTER A
TRIAL ENDS.
THIS INCLUDES FOR EXAMPLE
CONTINUED ACCESS TO THE DEVICE
ALREADY IMPLANTED.
FOLLOW-UP CARE RELATED TO THE
DEVICE.
AND DEVICE SUPPORT LIKE
MAINTENANCE HARDWARE REPLACEMENT
AND SOFTWARE UPDATES AND DEVICE
REPLACEMENT IN CERTAIN CASES,
THAT MAY NEED EMERGENCY CARE,
AND SIDE EFFECTS OR
COMPLICATIONS, AND POTENTIALLY
NEED FOR CARE COORDINATOR, AND
HEARD PATIENTS NAVIGATE PATHWAYS
TO FINDING CARE. AND MIGHT NEED
FOR PSYCHOLOGICAL SUPPORT, AND
NEED FOR STRONG, ADEQUATE
CONSENT, AND HEARD ABOUT
PATIENTS THROUGH RESEARCH
DESIGN.
AND HEARD ABOUT THE NEEDS FOR
REGISTRIES, WITH DEVELOPMENT
INFORMATION PROVIDERS COULD HAVE
ACCESS TO RELEVANT INFORMATION.
AND APOLOGIZE IF I'M SKIPPING
SOME NEEDS, BUT WE'LL WILL SURE
TO REGISTER ALL OF THEM AT A
LATER POINT IN TIME.
AND DURING THE SECOND PANEL WE
HEARD ONCE ALREADY BEING
COVERED.
IN PART, THE ANSWER THERE SOUNDS
LIKE IT DEPENDS.
THERE ARE A NUMBER OF THINGS
THAT ARE USUALLY COVERED BY
INSURANCE, INCLUDING EMERGENCY
CARE, OR EXPLANTATION IF IT'S
NEEDED FOR PROGRESSIVE
COMPLICATION OF THE DEVICE.
BUT IN MANY OTHER CASES,
DIFFERENT STAKEHOLDERS MAY OR
MAY NOT PROVIDE A CERTAIN TYPE
OF CARE DEPENDING ON THE CASE.
SOMETIMES IT'S A CASE BY CAY
BASIS, AND HEARD OF POLICIES
WHERE IT MAY FIT INSIDE OR
OUTSIDE OF THE POLICY.
I THINK FOR MANY OF THE TRIALS
NEEDS, WE HAVEN'T HEARD THAT
THERE IS CONSISTENT PROVISION OF
THIS CARE.
SO, I THINK THAT'S SOMEWHERE WE
WILL PICK UP TOMORROW, AS WE
START TO THINK ABOUT WHAT CARE
NEEDS SHOULD BE COVERED.
WHICH, I GUESS IS MY BRIDGE TO
TALKING ABOUT TOMORROW, WHERE WE
HOPE THAT ALL OF YOU WILL JOIN
US THEN.
WE'RE GOING TO HAVE ANOTHER TWO
SESSIONS IN THE AFTERNOON.
AND THE FIRST OF WHICH, IS GOING
TO COVER WHAT POST-TRIAL NEEDS
TO BE COVERED.
AND SECOND PANEL IS GOING TO
LOOK AT, OKAY, GIVEN THE
THINGS... GIVEN THE THINGS THAT
SHOULD BE COVERED -- SORRY ABOUT
THAT -- WHAT ARE THE GAPS IN
TERMS OF WHAT WE HEARD IN WHAT
IS CURRENTLY COVERED AND HOW CAN
WE BEST FILL THE GAPS, AND
HOPING THAT IS WHERE SOME OF THE
SOLUTIONS HAVE ALREADY BEEN
PROPOSED, AND MIGHT COME BACK
AND START TALKING ABOUT HOW WE
CAN APPROACH THEM, AND WHAT THE
NEXT STEPS COULD BE AND SO ON.
SO WITH THAT, I WANT TO ASK THE
CO-CHAIRS, AND THE EUROETHICS
WORKING GROUP IF THEY HAVE
SOMETHING ADDITIONAL TO ADD AND
I WANT TO AGAIN THANK YOU,
THANK YOU ALL FOR PARTICIPATING.
THANK YOU ALL FOR ATTENDING AND
I, I HOPE TO SEE YOU TOO,AND SEE
IF YOU HAVE SOMETHING TO ADD.
>> GREAT FIRST DAY, AND LOOKING
FORWARD TO TOMORROW.
>> PRETTY MUCH WHAT I WANTED TO
SAY, WONDERFUL PRESENTATION AND
DISCUSSION, AND LOTS TO THINK
ABOUT, AND TALK ABOUT TOMORROW
AS WELL.
THANK YOU ALL.
>> RIGHT WITH THAT WE CAN END
TODAY'S MEETING AND THANK YOU
ALL AGAIN, AND LOOKING FORWARD
TO SEEING YOU TOMORROW AND
THINKING ABOUT HOW TO RESOLVE
THESE COMPLICATED ISSUES.