>> THANK YOU.
WELCOME, EVERYBODY.
I APPRECIATE YOU JOINING US
TODAY.
I WANT TO WELCOME YOU TO THE
57th CTAC MEETING.
WE WANT TO WELCOME DR. DESTINE
SENIOR ADVISORY AND EXTERNAL
STRATEGY AND ONCOLOGY IN PLACE
OF DR. CASTER.
I'D LIKE TO READ THE OPENING
STATEMENT.
AS COMMITTEE MEMBERS, I WANT TO
REMIND YOU THAT WE MUST AB SENT
YOURSELF DURING SPECIFIC
DISCUSSIONS WHENEVER YOUR
PARTICIPATION OR DELIBERATIONS
ON A PRODUCT, PROGRAM OR OTHER
MATTER WOULD CONSTITUTE A
CONFLICT OF FROM OR CREATE THE
APPEARANCE OF CONFLICT OF
INTEREST.
IT'S INCUMBENT ON YOU TO ADVISE
THE EXECUTIVE SECRETARY FOR
ACTIONS REGARDING THAT MATTER.
IN LIGHT OF THE CURRENT POLICIES
REGARDING CONFLICT OF INTEREST
BASED ON FINANCIAL HOLDINGS OF
SPECIAL GOVERNMENT EMPLOYEES
INCLUDING ALL OF US ON THIS
COMMITTEE.
WE MUST DEPEND ON YOU TO
VOLUNTARILY ABSENT YOURSELF
DURING ANY AND ALL DISCUSSIONS
OF MATTERS THAT COULD
CONCEIVABLY IMPACT THE STATUS OF
THOSE HOLDINGS AND TRUST YOUR
JUDGMENT ON THESE INSTANCES.
BY LAW A QUORUM IS REQUIRED FOR
WHICH A VOTE OCCURS IN OPEN
SESSION.
DURING THIS MEETING A MINIMUM OF
10 APPOINTED MEMBERS MUST BE
PRESENT TO VOICE THEIR VOTES.
NEW MEMBERS WHO ARE NOT CURRENT
MEMBERS OF OTHER NCI BOARDS ARE
NEED TO BE CLEARED OF HHS OFFICE
AND HUMAN RESOURCES.
NOW I'LL READ THE PUBLIC
STATEMENT.
MEMBERS OF THE PUBLIC WHO MAY
WISH TO EXPRESS VIEWS REGARDING
ITEMS DURING THE MEETING MAY
WRITE THE EXECUTIVE SECRETARY,
SHEILA PRINDIVILLE WITHIN 10
DAYS AND WILL BE CAREFULLY
CONSIDERED.
THANK YOU.
THIS MEETING IS BEING BROADCAST
AND AVAILABLE TO THE PUBLIC AT
THE NIH VIDEOCAST WEBSITE AND
WILL BE ARCHIVED FOR VIEWING AT
A LATER POINT IN TIME.
I WANTED TO NOTE THAT THE FUTURE
MEETING DATES WERE SENT OUT TO
YOU.
THERE WERE THREE NEW DATES ADDED
FOR YOUR CALENDARS.
AND THOSE WERE SENT OUT PRIOR TO
THE MEETING.
A FEW OTHER REMINDERS.
PLEASE KEEP YOUR CAMERAS ON AT
ALL TIMES TO ENSURE THE MEETING
IS INTERACTIVE AS POSSIBLE.
USE THE RAISE YOUR HAND FEATURE
IN WEBEX TO ASK A QUESTION OR
COMMENT AND WE'LL TRY TO MAKE
SURE EVERYBODY GETS CALLED UPON
AND REMAIN MUTED DURING THE
WEBINAR UNLESS YOU'RE CALLED TO
SPEAK OR ASKING A QUESTION AND
THE RESPECTIVE PERSON WILL BE
CALLED UPON.
REMAIN PRESENT IN THE VIRTUAL
ROOM FOR THE FULL DURATION OF
THE MEETING.
A QUORUM OF BOARD MEMBERS IS
REQUIRED FOR ANY VOTE AND
SEVERAL VOTES ARE EXPECTED
DURING THIS BUSINESS MEETING.
BECAUSE OF THE TIMING OF THEIR
MOTIONS IT'S NOT PRE DETERMINED
YOUR FULL ATTENDANCE IS
REQUESTED DURING THE ENTIRE
MEETING.
ANY QUESTIONS ABOUT ANY OF THOSE
STATEMENTS?
ALL RIGHT.
NOW I'M GOING TO TALK ABOUT A
MOTION TO VOTE AND ACCEPT THE
SUMMARY MEETING FROM NOVEMBER 6
CTAC MEETING THE LAST ONE.
IS THERE A MOTION TO ACCEPT THE
SUMMARY?
>> SO MOVED.
>> A SECOND?
OKAY.
ANY DISCUSSION?
BY SHOW OF HANDS, PLEASE SEE WHO
WILL ACCEPT THE SUMMARY.
ANYONE WHO DOES NOT ACCEPT THE
SUMMARY OR ABSTENTIONS?
OKAY THE SUMMARY PRASS.
THANK YOU.
-- PASSES.
THANK YOU.
ANY OTHER DISCUSSION ITEMS
BRIEFLY?
ALL RIGHT.
THE NEXT ITEM IS DR. LOWY THE
DEPUTY DIRECTOR OF NCI WITH
BRIEF OPENING COMMENTS.
THANK YOU.
>> THANKS VERY MUCH.
GOOD MORNING, EVERYONE.
IT REALLY IS A PLEASURE FOR ME
TO BE HERE.
I'M GLAD CTAC IS CONVENING AT
SUCH AN IMPORTANT TIME FOR THOSE
WHO DO NOT KNOW I'VE BEEN
SERVING AS THE ACTING DIRECTOR
SINCE JANUARY 20th AND IT'S BEEN
A TUMULTUOUS TIME.
NOW MORE THAN EVER ADVISORY
BOARDS LIKE YOURS PLAY A VIOLATE
VITAL ROLE TO ENSURE WE REMAIN
RESPONSIVE TO THE NEEDS AND
PUBLIC AND RESEARCH COMMUNITY.
IN FACT OUR NUMBER OF EXTRAMURAL
COMMITTEES HAS GONE FROM FIVE TO
TWO INCLUDING CTAC.
THIS IS JUST ONE EXAMPLE OF MY
POWER.
A FEW MONTHS AGO NCI WAS ASKED
TO JUSTIFY THE NEED FOR EACH
COMMITTEE EXCEPT FOR NCAB AND WE
WROTE WHAT I THOUGHT WERE
SPIRITED DEFENSES OF THE FINLAC
AND NCRA AND CTAC COMMITTEES.
AND CTAC IS THE ONLY ONE OF
THESE LEFT STANDING.
I'M GOING TO DISCUSS SEVERAL
TOPICS TODAY.
UP FRONT FUNDING, NCAB WORKING
GROUP, CHIEF SCIENCE ADVISORY,
CANCER MORTALITY RATE TRENDS AND
THE COMMITMENT OF NCI STAFF.
BUT BEFORE I GET TO THOSE
TOPICS, I WANT TO TELL YOU ABOUT
A FEW IMPORTANT CHANGES WITH
DIRECT IMPACT ON CTAC.
FIRST, PAULETTE GRAY RETIRED AT
THE END OF JUNE.
AN ERA HAS END ED SHE DID SO MAY
THINGS.
DINAH SINGER HAS BECOME THE
DIRECTOR OF THE DIVISION OF
EXTRAMURAL ACTIVITIES AND SHAMLA
IS THE ASSOCIATE DIRECTOR FOR
SCIENTIFIC REVIEW AND POLICY.
I'D LIKE TO DEAL WITH THE FIRST
ISSUE IS THE UPFRONT FUNDING
POLICY AND I HAVE THREE SLIDES
ON IT.
SO IN JUNE THE OFFICE OF
MANAGEMENT AND BUDGET INFORMED
NIH EACH INSTITUTE MUST USE UP
FRONT FUNDING FOR AT LEAST 50%
OF ITS REMAINING RPG FUNDS.
NCI CAN CHOOSE WHICH AWARDS TO
DESIGNATE FOR UP FRONT FUNDING
PROVIDING THE TOTAL DOLLARS
REWARDED ARE 50% OR MORE OF THE
REMAINING RPG FUNDING.
SO WHAT IS UP FRONT FUNDING?
FUND FOR THE ENTIRE AWARD PERIOD
WHICH IS USUALLY FIVE YEARS ARE
OBLIGATED IN THE YEAR THE AWARD
IS MADE.
ALL THE FUNDS ARE MADE FROM THE
YEAR OF THE AWARD.
IT'S A SPECIAL CASE OF MULTIYEAR
FUNDING WHICH REFERS TO
OBLIGATING FUNDS FOR MORE THAN
ONE YEAR.
SOMETIMES A FIVE-YEAR AWARD WE
MIGHT MAKE TWO OR THREE-YEAR
AWARDS BUT NOW UP FRONT FUNDING
IS ALL FIVE YEARS AND INSTEAD OF
BEING LEFT UP TO NCI, THIS IS
WHAT WE ARE REQUIRED TO DO.
WE USUALLY HAVE JUST FUNDED ONE
YEAR AND THEN FUNDED EACH OF THE
SUBSEQUENT YEARS WITH YEAR BY
YEAR FUNDING.
I SHOULD POINT OUT THE LAST
BULLET POINT 50% UP FRONT
FUNDING IS INCLUDED IN THE
PRESIDENT'S PROPOSED BUDGET FOR
FY26 AND IT HAS BEEN SAID THAT
100% OF UP FRONT RPG FUNDING
WILL BE INCLUDED IN THE
PRESIDENT'S PROPOSED BUDGET FOR
FY27 ALTHOUGH THE BUDGET ITSELF
IS NOT SET PUBLIC.
THE NEXT IS A DIAGRAM I HAVE
MADE TO EXPLAIN THE DIFFERENCES
BETWEEN UM FRONT FUNDING AND
YEAR BY YEAR FUNDING.
AND UP FRONT FUNDING IN BROWN
AND ON THE BOTTOM YEAR BY YEAR
FUNDING IN GREEN.
I'D LIKE YOU TO FOCUS FIRST ON
YEAR ONE, WHICH I'VE OUTLINED
WITH THE OPEN RECTANGLE.
WITH YEAR BY YEAR FUNDING WHICH
IS WHAT WE ARE USED TO DOING AS
SHOWN IN THE GREEN, LET'S SAY WE
HAD ENOUGH MONEY WE COULD FUND
FIVE AWARDS FOR THAT YEAR.
THAT'S WHAT DEPICTED BY THE FIVE
GREEN RECTANGLES ON THE BOTTOM.
BY CONTRAST WITH UP FRONT
FUNDING IN YEAR ONE, YOU HAVE
THE SAME AMOUNT OF MONEY BUT YOU
NEED TO DEVOTE ALL OF IT TO ONE
AWARD.
THERE WOULD BE FIVE AWARDS BY
YEAR BY YEAR FUNDING AND ONE
AWARD WITH UP FRONT FUNDING.
OF COURSE FROM THE PERSPECTIVE
OF THE PEOPLE WHO GET THE UP
FRONT FUNDING, THEY ARE CERTAIN
OF HAVING THE MONEY THROUGHOUT
THE FIVE YEARS.
IN THE SECOND YEAR ASSUMING YOU
HAD THE SAME AMOUNT OF MONEY YOU
WOULD FUND ONE MORE AWARD WITH
UP FRONT FUNDING AND THE SECOND
YEAR WITH YEAR BY YEAR FUNDING.
THAT GOES ON THROUGH YEAR FIVE.
AND AT THE END OF YEAR FIVE, YOU
HAVE SPENT THE SAME AMOUNT OF
MONEY ON THE RESEARCH AND YOU
NOW HAVE FIVE YEARS -- FIVE
AWARDS YOU HAVE MADE BUT BECAUSE
OF WHEN THE AWARDS WERE MADE,
WITH YEAR BY YEAR FUNDING, THE
FIVE YEARS OF THE FIVE AWARDS
GIVE 25 RESEARCH YEARS OF
FUNDING.
ON THE OTHER HAND, IF YOU LOOK
ABOVE AT THE UP FRONT FUNDING
BECAUSE SO MUCH OF THE FUNDING
ACTUALLY GOES INTO YEARS SIX
THROUGH 10, YOU ONLY GET 15
RESEARCH YEARS OF FUNDING.
S BY THE TIME YOU GET TO THE
SECOND FIVE YEAR IN THE
HYPOTHETICAL EXAMPLE, BOTH GIVE
25 RESEARCH YEARS OF FUNDING BUT
IT TAKES A WHILE TO GET THERE.
THE NEXT SLIDE SUMMARIZES WHAT I
THINK WOULD HAPPEN.
ONCE STEADY STATE IS ACHIEVED UP
FRONT FUNDING AND YEAR BY YEAR
FUNDING RESULT IN THE SIMILAR
NUMBER OF AWARDS AND SIMILAR
NUMBER OF RESEARCH YEARS
CONDUCTED.
FOR FIVE YEAR AWARDS IT WOULD
TAKE FIVE YEARS OR MORE TO
ACHIEVE STEADY STATE.
THE SECOND BULLET EMPHASIZES
DURING THE RAMP UP PERIOD THERE
WOULD BE FEWER AWARDS INITIALLY
WITH UP FRONT FUNDING.
THE NUMBER OF RESEARCH YEARS
CONDUCTED WOULD BE FEWER THAN
WITH YEAR BY YEAR FUNDING DURING
THE RAMP UP PERIOD AND IT IS
REASONABLE TO SPECULATE THAT
THERE WOULD BE SUBSTANTIAL LOSS
OF PRINCIPLE INVESTIGATORS AND
STAFF.
I WANT TO TELL YOU NOW ABOUT A
NEW NCAB WORKING GROUP.
DURING THE MEETING THE BOARD
APPROVED THE ESTABLISHMENT OF A
NEW AD HOC RESEARCH GROUP ON
CONCEPTS AND PROGRAMS.
WE ENVISION THIS GROUP WILL TAKE
ON MANY OF THE RESPONSIBILITIES
PREVIOUSLY HANDLED BY THE NOW
RETIRED BOARD OF SCIENTIFIC
ADVISORS.
A DIRECT RELEVANCE TO CTAC, WE
INTEND FOR THE NEW WORKING GROUP
TO BE MADE UP OF A BROAD RANGE
OF CANCER RESEARCHERS AND
ADVOCATES.
THIS INCLUDES THE CURRENT
MEMBERSHIP OF CTAC.
THEN I'M PLEASED CTAC TODAY WILL
BE CONSIDERING THE ESTABLISHMENT
OF WORKING GROUPS ON STREAMLINE
DATA COLLECTION AND ADVANCING
INNOVATION AND STRENGTHENING THE
ROLE OF PATIENT PERSPECTIVE IN
CANCER RESEARCH.
NEXT IS SLIDE.
THE NEXT TOPIC IS THE CHIEF NEW
POSITION OF CHIEF SCIENTIFIC
ADVISER.
DR. GEORGE SEV OF -- DR
DR. SEGUNAS IS FROM EAST
CAROLINA UNIVERSITY AND
PRESIDENT DEN SL SHALLY
APPOINTED -- PRESIDENTIALLY
APPOINTED DIRECTOR AND WAS THE
FORMER DIRECTOR OF HRSA DURING
THE FIRST TRUMP ADMINISTRATION
AS THE HEALTH RESOURCES AND
SERVICES ADMINISTRATION.
AND BEFORE HE WENT TO EAST
CAROLINA UNIVERSITY, HE DID
SEVERAL YEARS OF POSTDOCTORAL
FELLOWSHIP AT NIH, DR. SIGOUNAS
HAS BEEN A REAL ASSET TALKING
ABOUT WHAT NCI IS TRYING TO DO
AND TRYING TO IMPROVE ON IT AND
BEING A LIAISON FOR US WITH THE
ADMINISTRATION.
DEF
>> I WANT TO TALK ABOUT CANCER
MORTALITY TRENDS.
THE GOOD NEWS IS SHOWN TO ME IN
THE UPPER GRAPH BECAUSE WE NOW
HAVE MORE THAN 30 YEARS OF
PROGRESSIVELY DECREASING CANCER
MORTALITY RATES IN THE UNITED
STATES BOTH FOR MEN AS WELL AS
FOR WOMEN.
YOU THIS MEANS 300,000 AMERICANS
WILL NOT DIE THIS YEAR FROM
CANCER WHO WOULD HAVE DIED IN
1990 MORTALITY RATES.
THAT'S TERRIFIC.
GONE DOWN BY MORE THAN
ONE-THIRD.
OF COURSE WE ARE ALL AWARE OF
THE 600,000 PATIENTS WHO ARE
GOING TO DIE OF CANCER DURING
THIS YEAR WHICH REALLY
EMPHASIZES THE URGENCY AND
IMPORTANCE OF WHAT WE NEED TO
DO.
THE NEXT SLIDE SIMPLY DEPICTS
FOR YOU DIFFERENT CANCER TYPES
WITH THE BLUE BEING THE ONES
WHERE WE'RE REDUCING MORTALITY
RATES AND THE RED BEING ONES
WHERE THE MORTALITY RATES ARE
CONTINUING TO INCREASE.
WHAT I HOPE YOU CAN APPRECIATE
IS THERE ARE MANY MORE FORMS OF
CANCER WHERE THE RATES OF
MORTALITY ARE GOING DOWN.
IF THIS HAD BEEN 15 YEARS AGO,
FOR EXAMPLE, MELANOMA AND LUNG
CANCER ARE CLOSE TO THE BOTTOM.
THEY'RE HAVING THE GREATEST
IMPROVEMENT WOULD NEVER HAVE
BEEN THERE 15 YEARS AGO.
SO IT'S A REALLY TESTIMONY TO
THE ADVANCES OVER THE RELATIVELY
RECENT PAST.
BUT THE NEXT SLIDE SHOWS YOU
THAT IN ADDITION TO THE 600,000
PEOPLE THAT WE NEED TO BE VERY
CONCERNED ABOUT THIS YEAR AND
GOING FORWARD, THE ADVANCES IN
CANCER RESEARCH HAVE NOT BEEN
UNIFORM WITHOUT THE UNITED
STATES.
THE DARK RED STATES IN
PARTICULAR REPRESENT THOSE
STATES WITH THE HIGHEST CANCER
MORTALITY RATES IN THE U.S.
EVEN THOSE STATES HAVE HAD A
DECREASE IN THE CANCER MORTALITY
RATES AND THE SOME HAVE
INCREASED SO THEY IN MOST OF
THESE STATES THEY'RE 20% OR
GREATER FOR THIS DISPARITY.
THE LAST OF MY COMMENTS IS I
JUST WANT TO REMIND ALL OF YOU
PEOPLE LIKE SHEILA PRINDIVILLE
AND JIM DORRSHELL AND OTHERS ARE
COMMITTED.
WE'RE EXTRAORDINARILY FORTUNATE
THOUGH WE'VE HAD ABOUT A 10%
REDUCTION IN NCI STAFF BETWEEN
VOLUNTARY SEPARATIONS AND IN
VOLUNTARY SEPARATIONS, THE
PEOPLE WHO ARE HERE ARE TOTALLY
COMMITTED TO OUR MISSION AND I
JUST WANT TO ASSURE YOU, WE WILL
BE DOING EVERYTHING WE CAN TO
HELP YOU BE ABLE TO ADVANCES AND
PROGRESS IN CANCER RESEARCH OUR
PATIENTS DESERVE NO LESS.
THE LAST SLIDE IS SIMPLY TO SAY
THANK YOU AND I LOOK FORWARD TO
YOUR QUESTIONS AND COMMENTS.
>> THANK YOU SO MUCH.
WE APPRECIATE THAT UPDATE ON
INFORMATION.
>> JULIE VOSE, I SHOULD HAVE
INCLUDED YOU.
APOLOGIES.
IT TAKES ALL OF US.
>> ABSOLUTELY.
THANK YOU SO MUCH.
SO ANY QUICK QUESTIONS FOR
DR. LOWY BEFORE WE MOVE FORWARD?
>> DOUG, I APPRECIATE ALL THE
EFFORTS OF YOURSELVES AND THE
FOLKS AT NCI DURING THIS
CHALLENGING PERIOD.
ONE QUESTION.
IT'S A NOT LOST ON ME WITH ALL
THE CHRONIC DISEASES HOW MANY
MORE OF OUR CANCERS COULD BE
THOUGHT OF IN THAT CATEGORY.
WE CLEARLY HAVE THE PRIOR
PARADIGM OF YOU HAD CANCER
EITHER YOU WERE CAREER OR YOU
ASPIRED FROM THE DISEASE.
WE HAVE MANY EXAMPLES NOW OF
PEOPLE WHO HAVE EXTENDED
SURVIVAL WITHOUT A CURE OR LIVED
WITH A CANCER IN A CHRONIC WAY.
IS THAT AN ONGOING DISCUSSION
JUST KEEPING THAT TOP OF MIND OF
HOW CANCER FITS IN THE SPECTRUM
OF CHRONIC DISEASE AS WELL AS
HOW CHRONIC DISEASES ARE CLEARLY
LINKED TO CANCER SUCH AS
OBESITY, THE METABOLIC SYNDROME,
ETCETERA.
>> YES.
I THINK IT IS WARREN TIBE ALSO
ON, ANOTHER DIRECTOR HERE, WE
TALKED WEEKS AGO WITH TWO PEOPLE
REPRESENTING THE MAHA EFFORT,
MAKE AMERICA HEALTHY AGAIN,
ABOUT NCI WHAT WE'RE DOING AND
CLEARLY UNDERSTAND.
I THINK WE FIND OURSELVES IN
GOOD NEWS, BAD NEWS.
GOOD NEWS, MADE A LOT OF
PROGRESS.
BAD NEWS, AFTER PLACES TO GO.
PATTY SPEARS.
>> FIRST, THANK YOU SO MUCH FOR
STEPPING UP TO THE PLATE.
YOU'RE ALWAYS THERE TO TAKE
CHARGE AND WE LOVE THAT.
I REALLY APPRECIATE YOU GOING
INTO WHAT THE UP FRONT FUNDING
WAS AND APPRECIATE THE PICTURE
BECAUSE IT WAS CLARIFYING FOR ME
WHAT THAT MEANS AND THERE MIGHT
BE THAT LULL WHEN THIS IS
INSTITUTED.
>> PATTY, IT IS NOT COPYRIGHTED.
>> I LOVE A GOOD PICTURE AND IT
WAS SO GOOD.
IF YOU MARRY THAT WITH POTENTIAL
CUTS IN FUNDING AND I'M WORRIED
ABOUT DELAYS IN RESEARCH AND
CLINICAL TRIALS AND LOSS OF OUR
WORKFORCE, OUR RESEARCHERS AND
THINGS.
USE THIS AS THIS NEEDS TO BE
USED IS WORRYING ME AS A PATIENT
GOING FORWARD.
>> TALKING WITH THE DIRECTORS
FROM THE COOPERATIVE GROUPS, I
GUESS ROB HAS HEARD THIS MORE
THAN ONCE.
WE ARE RESTRICTED IN TERMS OF
WHAT WE CAN SAY WHATEVER YOU
WANT.
>> RAHM.
>> THANK YOU ON BEHALF OF THE
CANCER RESEARCH COMMUNITY AND
NCI AND STAFF FOR WORKING HARD
TO KEEP US MOVING FORWARD.
OBVIOUSLY CHALLENGING TIMES.
I WANT TO HEAR YOUR THOUGHTS ON
HOW THIS UP FRONT FUNDING WOULD
CHANGE THE FUNDING OF GRANTS.
DO YOU SEE A BROAD EFFECT THAT'S
DISTRIBUTED ACROSS ALL PROGRAMS
OR DO YOU SEE PRIORITIZATION
FROM PROGRAM TO THE OTHER AS TO
HOW THIS WILL HIT THE RESEARCH
PROGRAMS?
>> THUS IS A GREAT QUESTION AND
WE DON'T HAVE A FULL ANSWER YET.
WE JUST STARTED WORKING WITH IT
AND WRESTLING WITH IT.
WHAT I SEE GOING FORWARD IN
2027 WHEN THE ADMINISTRATION IS
SAYING ALL RPG FUNDING IS GOING
TO BE UP FRONT IS THAT WE'RE
GOING TO BE LOOKING AT THE
SECOND OR THIRD PERCENTILE FOR
SUCCESS IF ALL WE DO IS GO BY
PEER REVIEW.
ONE THING WE DISCUSSED THIS
MORNING WAS HOW IS THAT GOING TO
SKEW THE CANCER RESEARCH
EFFORTS?
AND WHAT SHOULD WE BE DOING?
WE ARE STILL REALLY THINKING
ABOUT THAT.
I'D LIKE DO ASK, WARREN DID YOU
WANT TO ADD ANYTHING?
>> I DON'T THINK I NEED TO,
DOUG.
THAT WAS A GREAT ANSWER.
RAHM DID YOU HAVE SOMETHING ELSE
YOU WERE CONCERNED ABOUT?
>> NO, JUST WANTED TO SEE HOW
THIS WOULD IMPACT WHEN
IMPLEMENTED ACROSS PROGRAMS,
THAT'S ALL.
>> Dr. Lowy: SAY THE FIRST AND
SECOND PERCENTILE ARE ALL PEOPLE
DOING CLINICAL TRIALS OR SECOND
PERCENTILE ARE ALL PEOPLE DOING
BASIC RESEARCH.
WE CAN'T HAVE THAT HAPPEN.
AS IMPORTANT AS BASIC RESEARCH
AND CLINICAL TRIALS RESEARCH IS,
WE NEED TO TRY TO HAVE SOME KIND
OF BALANCE.
NOT EVERYONE WILL AGREE ON WHAT
THAT WOULD BE.
DR. AZAD.
>> DR. AZAD: FRANKLY I WANT TO
THANK ALL FOR ABOUT WHAT AS THE
BEING IN CHALLENGING TIMES AND
WE WANT TO FIGURE OUT WHAT WE
CAN DO AND I KNOW THERE ARE
CHALLENGES IN TERMS OF WHAT CAN
BE ASKED OF US AS WELL BUT I
DO -- I THINK WE'RE VERY READY
TO BE ORGANIZED IN WHATEVER WAY
WE CAN.
I WANTED TO ASK A QUESTION
FOLLOWING UP A LITTLE BIT ON THE
CONCERN ABOUT HAVING THE FUNDING
LINE HAS PARTICULAR
RAMIFICATIONS FOR OUR YOUNG
FOLKS AND FOR OUR EARLY CAREER
INVESTIGATORS AND K GRANTS AND
PART OF WHAT WE SHOULD BE
ADVOCATING FOR IS CARVE OUTS FOR
UP FRONT FUNDING TO HELP PROTECT
THEM AND THEY'LL BE HIT THE
HARDEST AND THE FUTURE OF OUR
ENTERPRISE.
CAN WE HELP WITH THAT?
>> Dr. Lowy: DR. AZAD, FOR RIGHT
NOW FY25, WE ARE TRYING TO AS IT
WERE, THINK ABOUT FUNDING A
LARGER PERCENTAGE OF EARLY STAGE
INVESTIGATORS THAN WHEN WE HAVE
BEEN FUNDING RECENTLY.
BUT THE TOTAL NUMBER OF EARLY
STAGE INVESTIGATORS WE'LL BE
FUNDING WILL BE SMALLER THAN THE
NUMBER WE HAVE BEEN.
WHERE WE MIGHT END UP IN '26 OR
'27 STILL REMAINS TO BE SEEN.
WE'RE HAVING A LOT OF ONGOING
DISCUSSIONS ABOUT IT.
LET ME REASSURE YOU THAT WE'RE
NOT JUST CONCERNED ABOUT EARLY
STAGE INVESTIGATORS WE'RE
CONCERNED ABOUT CANCER TRAINING.
PEOPLE BEFORE THEY GET THERE.
SO THESE ARE ISSUES WE CLEARLY
ARE CONCERNED ABOUT.
DR. WILDING.
>> WHAT WILL WILL BE HAPPENING
TO EVERYONE ELSE'S GRANT IN
THEIR SECOND OR THIRD YEAR?
>> Dr. Lowy: GREAT QUESTION.
WE ANTICIPATE TO CONTINUE THE
AWARDS AT A REASONABLE LEVEL I
MEAN ABOUT 95% OF THE
COMMITMENT.
OUR DIFFICULTY REALLY IS THAT WE
SUPPORT A TREMENDOUS AMOUNT OF
RESEARCH OUTSIDE THE RPG POOL.
I THINK WE'RE RELUCTANT TO TAKE
A LOT OF MONEY AWAY FROM THAT
BECAUSE WE'RE THEN
DISADVANTAGING THOSE AREAS.
IT IS A LOT EASIER TO ADD NEW
MONEY THAN IT IS TO TAKE AWAY
THE CURRENT LEVEL.
I DON'T THINK I'M TELLING YOU
ANYTHING YOU DON'T ALREADY KNOW.
>> THANK YOU SO MUCH.
I'M SORRY TO CUT THINGS OFF BUT
WE NEED TO MOVE ON TO THE NEXT
TOPIC.
>> Dr. Lowy: THANKS EVERYONE.
BEFORE I FINISH THOUGH, YOU ARE
ABSOLUTELY -- YOU AND THE
PATIENT ADVOCATES ARE THE BEST
PEOPLE WE HAVE TO TALK ABOUT THE
MIRACLE OF CANCER RESEARCH.
THE PEOPLE THAT WE HAVE WHO
SHOULD HAVE BEEN DEAD 10 YEARS
AGO AND ARE STILL ALIVE AND
WANTING TO GIVE EVERYONE THOSE
OPPORTUNITIES FOR THE NEW
RESEARCH TO TRY TO MAKE SURE
THAT WE CONTINUE TO MAKE THE
PROGRESS THAT OUR PATIENTS
DESERVE.
THANK YOU SO MUCH FOR WHAT
YOU'RE DOING.
>> THANK YOU.
THANK YOU FOR EVERYTHING YOU DO.
I THINK THE SLIDE YOU SHOWED IS
HELPFUL.
>> THEY'RE PUBLICALLY AVAILABLE
AS FAR AS I'M CONCERNED.
IF ANYBODY WANTS TO TALK WITH MK
HAL HOOLIMAN IS IN THE PUBLIC
AFFAIRS AND MAY BE ABLE TO HELP.
>> NOW WE'LL MOVE TO THE NEXT
TOPIC, DR. DOROSHOW WILL GIVE AN
UPDATE.
>> THANKS, EVERYBODY.
I'LL BE BRIEF.
IT'S GOING TO BE AN INTRODUCTION
TO TODAY'S AGENDA.
MANY HAVE PARTICIPATED IN OUR
EFFORT TO DEVELOP A STRATEGIC
PLAN FOR OUR CLINICAL TRIALS ON
MORE FLEXIBLE TRIALS AND TRIALS
THAT WE STREAMLINE THE PROCESS
FOR TRIAL EXECUTION, DECREASE
THE BURDEN AND FOCUS ON
ESSENTIAL END POINTS AND
INCREASE HOPEFULLY OUR
EFFICIENCY FOR DATA COLLECTION.
AND THERE ARE A VARIETY OF
THEMES.
MANY OF WHICH WE ELEMENTS
APPROACHES AND OPERATIONAL
BURDENS AND WE'LL TELL YOU THE
AGENDA TODAY IS REALLY AN
UPDATE.
WE'RE ABOUT FIVE YEARS IN AND WE
MADE A LOT OF PROGRESS AND SOME
OF THAT PROGRESS WILL BE
FOCUSSED AROUND THE
PRESENTATIONS FOR TODAY'S
MEETING.
WE'LL HAVE A PRESENTATION ON THE
AUDIT PROCESS.
WE'LL BE HEARING FROM MS.
DENICOFF ABOUT THE PRIORITIES
AND DATA COLLECTION.
MANY PARTICIPATED IN THE EFFORT
THAT'S TAKING ON STEAM AND HEAR
FROM JEFF ALLEN ABOUT A PROJECT
THEY'VE INITIATE AND WOULD LIKE
TO HEAR DISCUSSION AFTER HE'S
FINISHED ON WHAT WE SHOULD DO IN
THAT SPACE OR WHETHER WE SHOULD
WAIT UNTIL AFTER IT'S FINISHED
ITS JOB.
TO GIVE A BIRD'S-EYE VIEW OF THE
AGENDA.
IT'S ALL FOCUSSED ON TRYING TO
GET AN UPDATE ON THE
RECOMMENDATIONS FOR OUR
STRATEGIC PLANNING, HOW TO MAKE
CLINICAL TRIALS BETTER.
SO THANK YOU SO MUCH.
>> THANK YOU.
ANY QUESTIONS FOR JIM BEFORE WE
MOVE ON?
SEEING NONE, WE'LL HAVE A
DISCUSSION WITH JEFF ALLEN
FRIENDS OF CANCER REASON AND
TALKING ABOUT PRIVACY AND
CONTROLS AND MAYBE IF WE CAN DO
A PILOT AND WILL PRESENT WHAT
THEY'VE DONE TO DATE.
THANK YOU.
>> THANK YOU, FOR THE CHANCE TO
COME BRIEF THIS COMMITTEE ABOUT
A PROJECT THAT WE ARE GETTING
CLOSE TO GETTING UP AND RUNNING.
I'M THRILLED TO BE ABLE TO SHARE
UPDATES OF WHERE WE'VE COME AND
WHERE WE THINK WE'RE GOING TO BE
ABLE TO HEAD WITH THIS PROOF OF
CONCEPT PILOT.
AND VERY MUCH LOOK FORWARD TO
THE IDEAS THE COMMITTEE MAY HAVE
FOR US MOVING FORWARD.
BY WAY OF BACKGROUND ON THE NEXT
SLIDE, THIS EXTERNAL CONTROL ARM
PILOT IS REALLY A CONTINUATION
OF SOME WORK WE HAVE DONE OVER
THE LAST SEVERAL YEARS AROUND
THE POTENTIAL UTILIZATION OF
REAL WORLD EFFORTS AS A TOOL IN
RESEARCH.
OF COURSE I THINK THIS IN LARN
PART IS A TOPIC MOVING FORWARD
QUICKLY DUE TO THE TECHNOLOGICAL
ADVANCES AROUND DATA CAPTURE AND
ED IMPLEMENTATION AND
STANDARDIZATION OF ELECTRONIC
HEALTH RECORDS AND RECOGNIZED IN
THE LAST SEVERAL YEARS STARTING
WITH THE COMMITMENT BETWEEN THE
FDA AND THE BIO TECHNOLOGY
INDUSTRY THROUGH A RECENT
REAUTHORIZATION OF THE FDA USER
FEES TO SUPPORT PROJECTS AND
GUIDANCE DOCUMENTS AND MANY HAVE
GOTTEN UNDERWAY AND MANY HAVE
BEEN ISSUED IN THE LAST FEW
YEARS.
AT THE SAME TIME AS THAT WAS
GETTING GOING WE THOUGHT IT
WOULD BE HELPFUL RECOGNIZING THE
TECHNOLOGY COMPANIES THAT ARE
REALLY EXPONENTIALLY GROWING
OPPOSED TO WAITING FOR OTHER
PILOTS TO PLAY OUT GIVEN THE
UNIQUE INFRASTRUCTURE AVAILABLE
IN ONCOLOGY.
TO GIVE A BRIEF SUMMARY OF REAL
WORLD EVIDENCE PROJECTS THAT SET
THE FOUNDATION FOR THE PROJECT.
THE PAST PROJECTS DEPICTED IN
THIS TRAJECTORY OF TIME, THE
FIRST PILOT WAS A PARTNERSHIP
WITH ABOUT SEVEN DIFFERENT DATA
COMPANIES THAT HAVE DEVELOPED
THEIR OWN INTERNAL RESEARCH
DATABASES IN ORDER TO BEGIN TO
BRING INFORMATION TO DEATH AND
CURATE THE INFORMATION AND
BETTER UNDERSTAND HOW IT CAN BE
USED FOR BETTER RESEARCH IN
CLINICAL PRACTICE AND WHEN WE
CAN MOVE WITH THE DISPARATE DATA
SOURCES TO ALIGN ON A COMMON
PROTOCOL AND UNDERSTAND WHAT
INFORMATION COULD BE EXTRACTED
IN A UNIFORM WAY FROM DIFFERENT
SOURCES OF DATA.
IN THE SECOND PILOT BASED ON
SOME OF THAT INITIAL EXTRACTION
WE RECOGNIZED SOME TRADITIONAL
MEASURES IN CLINICAL TRIALS ARE
DIFFICULT TO EXTRACT FROM
ELECTRONIC HEALTH RECORDS.
THE DATA NEEDED TO BE DIFFERENT
TO THINK ABOUT THE OBSERVATIONS
THAT COULD BE MADE.
IN THIS PILOT WE EXPLORED A
PROXY END POINT LIKE TIME TO
TREATMENT DISCONTINUATION AND
TIME TO NEXT TREATMENT TO
UNDERSTAND THE EXTENT WHICH THEY
CORRELATE WITH MEASURES LIKE
OVER ALL SURVIVAL.
CERTAINLY THERE WERE CHALLENGES
ON DATA AND SUBSEQUENT THERAPY.
THE THIRD PILOT CALLED REAL
WORLD RESPONSE WAS WORKING WITH
THE DATA CURATORS TO UNDERSTAND
THE EXTEND A, IMAGES WERE
AVAILABLE OR RESPONSE RATES FROM
REAL WORLD DATA.
WE SAW IN MOST CASES IT WAS NOT
SCHEDULED SAME AS CLINICAL
TRIALS AND MOST DATA SOURCES IT
WASN'T EVEN AVAILABLE BUT WERE
ABLE TO EXTRACT FROM CLINICAL
NOTES FROM THE CLINICIANS TO BE
ABLE TO ESTIMATE RESPONSE IN A
CONSISTENT WAY ACROSS DATA SETS
BY IMPLEMENTING A PROTOCOL
SHOWING CONSISTENCY IN RESPONSE
RATES FOR THOSE TREATED IN
CHEMOTHERAPY IN THIS PILOT
SETTING THE STAGE TO NOT JUST
WHAT THE INDEPENDENT DATA SETS
CAN DO AS THEIR OWN RESEARCH
DATA TOOLS BUT AUGMENT CLINICAL
TRIALS.
THIS IS HOW WE ARRIVED AT THE
EXTERNAL CONTROL ARM PROJECT
THAT I'LL WALK THROUGH A LITTLE
BIT TODAY.
ON THE NEXT SLIDE I WON'T SPEND
A LOT OF TIME ON THIS PARTICULAR
BACKGROUND BUT I THINK THE
CHALLENGES HERE ARE PRETTY WELL
CHARACTERIZED.
A RANDOMIZED CONTROL ARM CAN BE
DIFFICULT IN CERTAIN
CIRCUMSTANCES TO IMPLEMENT
BECAUSE OF THE RARITY OF THE
CONDITION.
IT MAY JUST BE VERY DIFFICULT TO
HAVE ENOUGH PATIENTS IN ORDER TO
SUFFICIENTLY RANDOMIZE THE TRIAL
IN A SUFFICIENT TIME FRAME AND
MADE ETHICAL CONSIDERATIONS IN
TERMS OF THE AVAILABILITY OF
THERAPY OF HAVING TO RANDOMIZE
TO A LESS EFFICACIOUS THERAPY OR
KNOWN THERAPY AVAILABLE.
THE IDEA OF USING THE EXTERNAL
CONTROL ARM IS TO BRING IN DATA
FROM AN EXTERNAL SOURCE, REAL
WORLD DATA USING PATIENT
RECORDS, INSURANCE CLAIMS,
PROSPECTIVE DISEASE REGISTRIES
OR CONSTRUCT A VIRTUAL CONTROL
GROUP IN ORDER TO PROVIDE
ADDITIONAL CONTEXT TO
INTERVENTIONAL SINGLE ARM STUDY.
NOW, THE IDEA ISN'T NEW.
CERTAINLY OTHER RESEARCHERS HAVE
DEVELOPED PROTOTYPES FOR
BUILDING THE EXTERNAL CONTROL
ARM IN THE PAST.
BUT WHAT WE'RE HOPING IS TO
DETERMINE OPTIMAL
CHARACTERISTICS OR THE CRITERIA
NECESSARY FOR SOURCES DATA IN
ORDER TO HELP IMPROVE THE
CONFIDENCE AND ACCEPTANCE OF
THEIR USE IN CONTROL.
THE UNIQUE ASPECT OF WHAT WE ARE
TRYING TO BUILD IS BUILD
EXTERNAL CONTROL ARMS TO
UNDERSTAND THE CHARACTERISTIC OF
THE DATA OF DIFFERENT SOURCES
AND UNDERSTAND THE EXTENT TO
WHICH WE CAN REPLICATE TO
IMPROVE THE CONFIDENCE.
SO IN THE NEXT SLIDE, I'VE LAID
OUT SOME OF OUR OVER ALL
OBJECTIVES FOR THIS PROJECT.
THE FIRST GOAL IS TO CONSTRUCT
AN ECA USING PATIENT-LEVEL DATA
FROM DIFFERENT DATA SOURCES THAT
LEVERAGING THE KNOWN PROGNOSTIC
AND DEMOCRATIC CHARACTERISTICS
FROM A CLINICAL TRIAL ARE ABLE
TO MATCH AT A PATIENT LEVEL TO
THE CONTROL ARM OF A RANDOMIZED
CONTROL ARM TRIAL OR THE TARGET
TRIAL.
AND THROUGH THIS WE HOPE TO
ESTABLISH A METHODOLOGY OF A
PROCESS THAT MIGHT BE ABLE TO BE
IMPLEMENTED MORE READILY IN THE
FUTURE TO AUGMENT EXISTING OR
UPCOMING RANDOMIZED CLINICAL
TRIALS.
WE RECOGNIZE THE FDA HAS ISSUED
GUIDANCE DOCUMENTS ON EXTERNAL
CONTROL ARMS IN 2023 TO HELP LAY
OUT CONSIDERATIONS FOR THE
RESEARCH COMMUNITY ABOUT THE
SOURCES OF DATA AND METHODOLOGY
APPLIED BUT THUS FAR HAS BEEN
SELDO
SELDOMLY IMPLEMENTED AND WE HOPE
THE PROJECT PILOT WILL HELP
RAISE THE DATA QUALITY OF THE
DATA SOURCED AND ULTIMATELY
PROVIDE ADDITIONAL GUIDANCE AS
TO HOW THESE TOOLS COULD BE
USED.
SO WE HOPE FROM A REGULATORY
STANDPOINT WE'LL BE ABLE TO
ESTABLISH CRITERIA WHETHER
DIFFERENT DATA SOURCES THAT MAY
BE E.H.R. DERIVED REAL WORLD
DATA OR PILOT CONTROL AND HAVE
DATA FROM BOTH SOURCES AND
UNDERSTAND WHETHER THEY'RE FIT
FOR PURPOSE FOR DEVELOPING AN
ECA.
WE HOPE WE'LL BE ABLE TO
IDENTIFY CHARACTERISTICS OF THE
DATA SOURCE THAT MAKE IT MORE
ACCEPTABLE IN ORDER TO BE ABLE
TO REPLICATE THE ELIGIBILITY AND
THE TIMING MEASURES INCLUDED IN
A RANDOMIZED CLINICAL TRIAL TO
MAKE THE ECA MORE FEASIBLE AND
METHODOLOGIES TO INFORM BEST
PRACTICES IN THE FUTURE.
IN THE NEXT SLIDE THE SITUATION
WE WERE LOOKING FOR IN ORDER TO
TRY AND IDENTIFY A TARGET TRIAL
TO REPLICATE WE SELECTED A
CONTROL EXAMPLE WHERE VARIOUS
FACTORS WERE SIMILAR TO A
SCENARIO WHERE AN EXTERNAL
CONTROL ARM MAY BE ACCEPTABLE
AND WANTED TO HAVE A PATIENT
POPULATION OR AREA WHERE IT WAS
RELATIVELY SMALL ENOUGH AN ECA
COULD BE THOUGHT ABOUT AS A
COMPONENT OF CLINICAL RESEARCH
BUT STILL FEASIBLE A CLINICAL
TRIAL COULD BE REPLICATED.
WE LOOKED AT METASTATIC CANCER
WITH THE UNMET NEED IT PRESENTS.
IMPORTANTLY IN THIS CASE THE
STANDARD OF CARE HAS REMAINED
RELATIVELY UNCHANGED OVER TIME.
THE MAJORITY OF THE DATA
INCLUDED IN THIS IS
RETROSPECTIVE TO DATA FROM
ELECTRONIC HEALTH RECORDS.
WE NEEDED SOMETHING THAT HAD NOT
BEEN RAPIDLY EVOLVING IN ORDER
TO USE HISTORIC DATA FOR THIS
USE.
WE CHOSE THE RESOLVE TRIAL AS
THE TARGET TRIAL.
WE'VE OBTAINED THE DATA AND IT
WAS A TRIAL THAT EVALUATED THE
SAFETY AND EFFICACY OF IBRUTNIB
AND NAB-PACLITAXEL AND IT'S THE
CONTROL ARM TO REPLICATE FOR
THIS PARTICULAR PILOT PROJECT.
AND TO UNDERSTAND WHETHER OUR
DATA SOURCES HAVE THE CAPABILITY
TO CONSTRUCT THE ECA WE ASKED
EACH POTENTIAL DATA PARTNER TO
ISOLATE THE PATIENT IN THEIR
BASE AND PULLED THE DATA FROM
IMPLEMENTING A COMMON PROTOCOL
TO APPLY TO THEIR DATA AND
UNDERSTAND THE NUMBERS AND
AVAILABLE PATIENT DATA FROM A
FEASIBILITY STANDPOINT TO FIT
THE CRITERIA OF THE TARGET TRIAL
CONTROL ARM.
ON THE NEXT SLIDE THIS BRIEFLY
DESCRIBES THE NUMBERS WE
OBSERVED FROM SIX DIFFERENT DATA
SETS THAT HAVE SUBMITTED THEIR
DATA THUS FAR.
AS YOU CAN SEE FIVE ARE BEING
UTILIZED ON REAL WORLD DATA FROM
ELECTRONIC HEALTH RECORDS THEY
CURATED FOR RESEARCH PURPOSES
AND ONE LEVERAGING PRIOR
CLINICAL TRIAL DATA THEY ASKED
OF THEIR SYSTEM OVER THE LAST
YEARS.
THE RANGE AND ELIGIBLE PATIENTS
THAT FIT THE CRITERIA OF THE
TYPE OF CANCER AND CONTROL ARM
TREATMENT THAT WE'RE LOOKING AT
RANGE FROM 39 TO OVER 2600
PATIENTS IN DIFFERENT DATA SETS
AND A MEDIAN ACROSS THE GROUPS
OF 326.
IN ORDER TO CONSTRUCT THE
EXTERNAL CONTROL ARM WE HAVE A
STATISTICAL ADVISORY GROUP THAT
HAS HELPED US PREPARE THE
STATISTICAL ANALYSIS PLAN FOR
THE PROJECT AND I WON'T GET OVER
MY STATISTICAL SKIS HERE EXCEPT
TO SAY THE MINIMUM DESIRABLE
NUMBER IS 171 PATIENTS FOR EACH
IS 80% OF THE TOTAL CONTROL ARM
IN ORDER TO BE ABLE TO DO
SUFFICIENT MATCHING BY
PROPENSITY SCORES BASED ON THE
CRITERIA OF THE TRIAL IN ORDER
TO BE ABLE TO RUN THE ANALYSIS.
IF IT'S LESS THAN THAT WE HAVE
COME UP WITH A PLAN FOR THOSE
WHO MAY HAVE FEWER DATAED TO
COMPARE THE DIFFERENCES IN THE
OUTCOMES FROM EACH OF THE
DIFFERENT ECAs COMPARED TO THE
TARGET TRIAL.
SO, ON THE NEXT SLIDE, AS THE
NEXT STEP TO ISOLATING THE
PATIENT POPULATION TREATED
SIMILAR TO THE RESOLVE TRIAL, WE
AIM TO ALIGN TO THE INCLUSION
AND EXCLUSION CRITERIA AND THE
EXCLUSION IS ON THE LEFT FROM
THE RESOLVE TRIAL AND THE
CORRESPONDING NUMBERS FOR THE
DATA SETS SUBMITTED THUS FAR ARE
DISPLAYED AAS S -- AS THE
INCLUSION AND EXCLUSION.
THIS APPLIES TO THE MISSINGNESS
OF DATA.
IN SUMMARY YOU CAN SEE THAT AS
MORE EXCLUSIONARY CRITERIA ARE
APPLIED THE NUMBER OF PATIENTS
AVAILABLE TO FIT TO CORRESPOND
WITH THE TARGET TRIAL FOR EXACT
MATCHING GOES DOWN.
THE GOOD NEWS IS THAT FROM EACH
OF THE SIX DIFFERENT DATA
PARTNERS LISTED HERE ONLY TWO OF
THEM HAVE THE DESIRED 171
NECESSARY IN ORDER TO DO THE
DIRECT PROPENSITY SCORE
MATCHING.
SO FOR THOSE TWO THAT HAVE UNDER
171, WE ARE STILL IN THE PROCESS
OF EXPLORING THE OPTIMAL
APPROACH OF RUNNING THE ANALYSES
AND STILL BE ABLE TO LEVERAGE
THE DATA BY WEIGHTING SOME OF
THE DATA THERE OR PERHAPS LO
LOOSENING THE DATA SET FOR WHICH
THEY OBSERVED A SIGNIFICANT
AMOUNT OF ATTRITION.
THOUGH WE KNOW WE'VE INTRODUCED
VARIABILITY WE THINK IT WILL
HELP UNDERSTAND WHETHER THE
EXTENT OF MISSING THE DATA
ACCEPTABLE TO ACCURATELY
CONSTRUCT AN ECA OR THE CRITERIA
NEEDED FOR SOURCE DATA TO BE
ULTIMATELY FIT FOR PURPOSE TO BE
AN ECA BY OBSERVING THAT
VARIABILITY BETWEEN DATA SOURCES
THAT HAVE DIFFERENT
CHARACTERISTICS OF DATA
AVAILABLE.
SO ON THE NEXT SLIDE IS AN
UPDATE OF WHERE WE ARE.
WE HAVE -- BEFORE I GET TO THE
UPDATE OF WHERE WE ARE, THIS IS
THE SUMMARY OF ANALYSES WE'LL BE
DOING AS WE CONSTRUCT THIS.
THIS IS HOPEFULLY WHAT THE TOTAL
PROJECT WILL BEGIN TO LOOK LIKE.
THE TOP BOXES ARE REPRESENTING
THE CLINICAL TRIAL SOURCE DATA.
THE DATA WE'LL BE USING CASE
REPORT FORMS FROM HISTORIC
CLINICAL TRIALS TO REPLICATE
THEIR ECA.
WE OBSERVE THERE'LL BE LESS
MISSING AND MORE THAN AN EHR
SOURCE WOULD HAVE AND FOR THOSE
ADDITIONAL FIVE EHR SOURCE ECAs
WE'LL BE CONSTRUCTING AN
INDIVIDUAL CONTROL ARM WHERE
EACH DATA PARTNER WILL HAVE
PROPENSITY SCORE MATCHING TO
MATCH THE PATIENTS IN THE
RESOLVE TRIAL AND IN THE
ANALYSES RECONSTRUCTING AND
LOOKING AT THE KM CURVES AND
HAZARD RATIO FOR THE TARGET
CONTROL AND COMPARING THE SIX
DIFFERENT CONTROL ARMS IN THE
PILOT WE HOPE WE'LL BE ABLE TO
OBSERVE THE AVAILABILITY FROM
DIFFERENT SOURCE DATA WHETHER
THE E.H.R.s OR CLINICAL TRIAL OR
DIFFERENT ALIGNMENT TO THE
ELIGIBILITY CRITERIA OF THE
ORIGINAL TRIAL.
THIS JUST GIVES A SUMMARY OF
WHERE WE ARE.
SO WE HAVE COMPLETE THE SCOPING.
WE KNOW THE NUMBER OF DATA
SOURCES THAT WILL BE ABLE TO
PARTICIPATE AND THE BROAD
WORKING GROUP AND THE
STATISTICAL WORKING GROUP HAS
COMPLETE THE PILOT DESIGN.
WE'VE IDENTIFIED THE STUDY
POPULATION WITH THE INCLUSION
AND EXCLUSION CRITERIA AND
CONTRACTING FOR THE DATA FROM
THE TARGET TRIAL IN ORDER TO GET
THE STUDY UP AND RUNNING AND OUR
PLAN IS FOR THE DURATION OF THIS
YEAR BE ABLE TO OVER THE NEXT
SEVERAL MONTHS CONDUCT THE
STATISTICAL ANALYSIS TO APPLY
THE COMMON PROTOCOL TO DATA AND
THEN TAKE THE POPULATIONS THEY
ARE CONSTRUCT AND COMPARE THE
OUTCOMES TO THAT OF THE TARGET
TRIAL AND DIFFERENT PARTICIPANTS
IN THE PILOT PROJECT AND OUR
INTENTION IS TO REPORT THE
FINDINGS FROM THIS IN THE FIRST
PORTION OF 2026.
IN THE FINAL SLIDE I WANT TO
THANK EVERYONE WHO HAS HELPED
PROVIDE EXPERTISE AND GUIDANCE
FOR THIS PROJECT OVER THE LAST
YEAR OR SO TRYING TO CONSTRUCT
IT AND IN PARTICULAR THOSE WHO
HAVE THE INTENTION OF RUNNING
THE ANALYSIS IN THE COMING
MONTHS.
I'LL STOP THERE.
THANK YOU FOR THE OPPORTUNITY TO
SHARE SOME OF THE EARLY WORK AND
LOOK FORWARD TO SHARING RESULTS
HOPEFULLY SOON BUT IF YOU HAVE
QUESTIONS OR IDEAS THE COMMITTEE
HAS TODAY.
>> THANK YOU, THAT'S VERY
AWESOME.
WE HAVE TIME FOR A FEW
QUESTIONS.
>> THANK YOU AND YOUR
PRESENTATION CLEARLY LAID OUT
HOW YOU SELECT THE CONTROL GROUP
FROM USING BASELINE ELIGIBILITY,
EXCLUSION CRITERIA SO THAT'S
GREAT.
MY QUESTION IS HOW DO YOU
CONTROL FOR WHAT HAPPENS BEYOND
THAT DURING THE COURSE OF
TREATMENT.
FOR INSTANCE, IF I'M TREATING A
PATIENT IN A SITUATION I HAVE
THE LUXURY OF IF THE PATIENT
MEETS RADIATION, I CAN DO A DOSE
AND GO BACK TO THE SAME
TREATMENT BUT AS THE CLINICAL
TRIAL DOES NOT HAVE THOSE --
LIKE THE PATIENT WANTS TO GO ON
VACATION FOR THREE WEEKS.
I CAN DELAY TREATMENT FOR THREE
WEEKS, NO PROB.
HOW DO YOU CONTROL FOR WHAT
HAPPENS DURING THE COURSE OF
TREATMENT SO THE COHORT MATCHES
AS CLOSE AS POSSIBLE?
>> THERE'S A NUMBER OF DATA
SOURCES BASED ON CLINICAL
PRACTICE.
SOME DISCUSSIONS WE HAD AT FDA
AS WELL AND RECOGNIZE A
PROSPECTIVE REGISTRY COLLECTION
OF DATA FROM CLINICAL PRACTICE
WOULD BE BETTER OFF THAN A
RETROSPECTIVE REAL WORLD DATA
SET BUT FROM OUR VANTAGE POINT
WE THOUGHT WHILE THAT MAY BE THE
CASE, LET'S PRESSURE TEST IT AND
SEE HOW DIVERGENT THE DIFFERENT
DATA SOURCES ARE.
HOW SIMILAR THE PATIENT
POPULATIONS CAN BE BY TRYING TO
SET UP CRITERIA AND MAYBE WE
WILL FIND THE REAL WORLD DATA IN
ITS CURRENT FORM IS NOT
SUFFICIENT TO REPLICATE A
CLINICAL CONTROL ARM OF THE
TRIAL BUT MAYBE IN THIS SCENARIO
WHERE THERE ARE FEW TREATMENT
OPTIONS WHERE UNFORTUNATELY THE
OUTCOMES ARE RELATIVELY SHORT
HERE THAT MAYBE SOME OF THE
VARIABILITY THAT CAN BE
INTRODUCED IN OTHER SETTINGS MAY
NOT BE IN THIS PLACE AND THIS
COULD BE ANOTHER INDICATOR AS TO
WHEN AN ECA MAY BE APPROPRIATE
TO USE.
>> I THINK IT'S A GOOD THING TO
HAVE A REAL WORLD EC A AND IT'S
ALMOST A DIFFERENT KIND OF
CONTROL NO SO MANICURED, IF YOU
WILL.
>> WE WANT TO MAKE SURE THEY'RE
VETTED FOR THAT OR PERHAPS IF
YOU HAVE BIG DATA SETS YOU CAN
HAVE MORE HYPOTHESIS DRIVEN
QUESTIONS ON GENERATING
POPULATION SPECIFIC EXTERNAL
CONTROLS AND LOOKING AT
DIFFERENCES THAT MAY BE VALUABLE
THAT WE DON'T HAVE AN
OPPORTUNITY TO DO AS WE DESIGN
THE RANDOMIZATION.
>> THAT'S AN IMPORTANT POINT.
I DON'T WANT TO GET AHEAD OF OUR
DATA PARTNERS AND CAUSE
ADDITIONAL WORK BUT THE DATA
THEY'LL BE ISOLATING WILL BE A
1:1 PATIENT MATCH FOR THOSE IN
THE TRIAL.
SO, BY DESIGN IT WILL HOPEFULLY
LOOK SIMILAR TO THAT COHORT FOR
THE TRIAL BUT WHAT WOULD BE
INTERESTING TO YOUR POINT WOULD
BE COULD WE ALSO TAKE THE SAME
DATA SET AND EVALUATE THE
PATIENTS PART OF THE ECA VERSUS
THOSE THAT WEREN'T ASSUMING
PERHAPS THEY CLEARLY HAVE
DIFFERENT CHARACTERISTICS THAT
DIDN'T ALLOW THEM TO BE A DIRECT
MATCH TO THOSE IN THE TRIAL AND
UNDERSTAND WHETHER THERE ARE
SIGNIFICANT DIFFERENCES IN THE
OBSERVATIONS BETWEEN THAT
CLINICAL TRIAL POPULATION AND
THE ACTUAL REAL WORLD POPULATION
THAT WE SEE IN THE TREATMENT.
IT'S A GREAT IDEA.
>> PATTY, I THINK YOU'RE NEXT.
>> I THINK THIS IS A GREAT
PROJECT.
I THINK THIS IS REALLY NEEDED.
IT COMES UP ALL THE TIME IN ALL
DIFFERENT AREAS.
I THINK IT'S REALLY ABOUT
BUILDING THAT CONFIDENCE SO THAT
PEOPLE WILL ACTUALLY USE THE
EXTERNAL CONTROL ARMS AND THIS
IS A GREAT PILOT BECAUSE IT IS A
CANCER STANDARD TREATMENT THAT
HASN'T CHANGED AND SOME OF THE
AREAS TREATMENT IS CHANGING SO
RAPIDLY IT'S GOING TO BE REALLY
HARD BUT I THINK WE CAN TALK
ABOUT THAT WHEN IT'S NEEDED.
THE QUESTION I HAD IS WHEN
YOU'RE DOING THE COMPARISON, HOW
CLOSE DO YOU NEED TO BE TO BE
SUCCESSFUL.
WHAT IS SUCCESS GOING TO BE
DRAWN BY TO BUILD THE CONFIDENT
YOU'RE NOT LOSING ANYTHING --
>> THAT'S A GREAT QUESTION.
SO WE HAVEN'T REALLY QUITE YET
DETERMINED HOW IMPORTANT IS C
CONCORDANT ENOUGH.
I SUPPOSE IT WILL BE IN THE EYE
OF THIS BEHOLDER AND WE'LL USE
WHAT WE CAN TO INFORM THE
METHODOLOGY.
WOULDN'T IT BE GREAT IF THE ECAs
WERE CLOSE TO THE TARGET AND THE
THING ABOUT THE PILOT IS A
LOW-RISK PILOT.
WE'RE NOT ADJUDICATING A PAST
CLINICAL TRIAL.
WE'RE TRYING TO SEE CAN IT WORK?
WE HAVE SIX OR SEVEN DATA
SOURCES FROM DIFFERENT PLACES
AND HAVE DIFFERENT DEMOGRAPHICS
WE'RE TRYING TO IMPLEMENT A
COMMON PROTOCOL JUST TO SEE WILL
IT WORK.
IT'S NOT QUITE AS EXCITING AS
USING AN ECA I SUPPOSE, BUT IT'S
A STEPPING STONE TO THINK ABOUT
THE RIGHT METHODOLOGY AND
THEY'RE APPROPRIATELY FIT FOR
PURPOSE FOR THE USE.
IF THEY ARE, GREAT, IF NOT WHAT
CAN THEY DO TO GET THERE IN THE
NEAR FUTURE.
>> ADAM, YOU'RE NEXT.
>> THAT WAS FANTASTIC.
I HOPE WE CAN SHAVE YEARS OFF
GETTING A GREATER NUMBER OF
ORPHAN DISEASES TO APPROVAL.
HOW DOES CONTROL ARM OF THE
STUFF IN THE DATA SPHERE, CAN
YOU USE IT IN THE GLOBAL EFFORT
OF YOURS?
>> IT'S A GOOD QUESTION.
WE HAVE BEEN WORKING WITH
PROJECT DATA SPHERE ON A
SLIGHTLY DIFFERENT PROJECT
AROUND THE IMAGING DATA THEY
HAVE.
MY UNDERSTANDING IS THAT AT
LEAST FOR THE THERAPEUTIC AREA
WE SELECTED FOR THE PILOT THEY
DON'T HAVE THE DATA THAT COULD
BE DRAWN FROM IN ORDER TO
SUFFICIENTLY MATCH IN THE
METASTATIC SETTING AND THE
REASON WE DID THAT I THINK WE
DID HAVE THOUGHT THAT IT MAY BE
GOOD METHODOLOGY BUT NOT GOOD
FOR MATCHING FOR A MORE LIMITED
DATA SOURCE AND WHY WE CHOSE THE
SCENARIO WE DID.
I'D LIKE TO THINK IF WE OR OTHER
PILOTS ARE ACCESSIBLE IT'S FOR
SHARING OF DATA THAT WOULD MAKE
THIS MORE READILY AVAILABLE
BECAUSE WE WOULDN'T WANT TO DO
THIS SEVEN TIMES OVER FOR ONE
ECA.
YOU'D LIKE ONE MAYBE TWO TO
VERIFY BUT A FIT FOR PURPOSE
RELIABLE DATA SET YOU COULD
LEVERAGE IN ORDER TO AUGMENT THE
CLINICAL TRIAL.
>> KEN AND THEN WE'LL HAVE TO
MOVE ON.
KEN I THINK YOU'RE MUTED.
>> I WANT TO SAY JEFF, I STUDY
RARE DISEASES MYELOFIBROSIS AND
THIS IS A CRUCIAL NEED.
WE'RE IN A POSITION WHERE WE HAD
A DECADE OF REALLY ONE ONLY
APPROVED DRUG AND THEN THE PHASE
III TRIALS THAT ARE TRYING TO
COMPARE AGAINST THE DRUG
STRUGGLED BECAUSE IT'S SUCH A
WIDELY AVAILABLE STANDARD.
HAVING ACCESS TO REAL WORLD DATA
WHETHER IT'S A GLOBE OF DATA
REGARDING THE POPULATION ALREADY
EXISTS.
I THINK AS WE TRY TO WEAVE IN
THE INFORMATION IN TERMS OF
LONGER FOLLOW-UP SURVIVAL AND
OTHER THINGS IT COMPLICATED
HAVING CONTROL ARMS AND THESE
REAL WORLD EVIDENCE CONTROL ARMS
WILL BE KEY FOR SOME OF THESE
WHERE IT'S VERY UNAC --
[INDISCERNIBLE] TO BE ON THE
CONTROL ARM.
>> ONE THING OF INTEREST TOO IS
WE HAVEN'T DETERMINED IF IT'S
GOING TO BE PART OF OUR ANALYSIS
MAYBE AS A STEPPING ZONE A
RANDOMIZATION EXTREME WHERE A
COMPONENT MAY BE
CONTEMPORANEOUSLY CONTROLLED AND
HALF ARE FROM A REAL WORLD DATA
SOURCE TO AUGMENT THE CLINICAL
TRIAL TO YOUR POINT MOST
PATIENTS LOOKING TO ENROLL IN A
CLINICAL STUDY ARE OFTEN TIMES
DOING SO FOR POTENTIAL ACCESS TO
THE INTERVENTIONAL ARM OR
EXPERIMENTAL ARM.
HOW CAN WE RECOGNIZE THAT AND
TRY TO MAKE THE STUDIES A LITTLE
BIT MORE FEASIBLE TO PARTICIPATE
IN.
>> WHEN WE TALK ABOUT EXTERNAL
CONTROL ARMS EVERYONE THINKS OF
REGULATORY APPROVAL PURPOSES
WHICH IS GREAT BUT AS I THINK OF
THE PRESENT, THIS COULD BE A
FANTASTIC RESOURCE FOR TRIAL
PLANNING TO TRY TO REDUCE THE
FAILURE RATE OF OUR PHASE III
TRIALS.
THE SECOND IS THAT IN PLANNING
POSTAPPROVAL ACTIVITIES AND TRY
TO EXTRAPOLATE A DRUG INTO AN
EFFECTIVE TREATMENT.
>> THANKS SO MUCH.
SO AS WE TALKED ABOUT BEFORE, WE
WERE PLANNING ON KIND OF DOING
THE SAME SORT OF ACTIVITY BUT
GIVEN THE FACT THAT FRIENDS OF
CANCER RESEARCH ARE WELL INTO
THAT SHOULD WE DELAY THE
ACTIVITY UNTIL WE CAN POST
RESULTS WHICH WOULD BE PRUDENT
FROM THIS STANDPOINT.
BUT ANY DISCUSSION OB -- ON
THAT?
>> AGREED.
>> WE CAN TALK ABOUT THAT LATER.
THANK YOU SO MUCH.
WE APPRECIATE THE PRESENTATION
AND AWESOME WORK.
THANK YOU.
ALL RIGHT.
MOVING ON TO THE SECTION HERE ON
STREAMLINING CLINICAL TRIAL
IMPLEMENTATION IN OUR UPDATE.
DR. JIM DOROSHOW WILL NOW GIVE
AN UPDATE ON THAT.
>> WE GOT SPECIFIC
RECOMMENDATIONS FROM OUR
PLANNING GROUP AND WHEN WE
REVIEWED THESE WAS COULD WE
REDESIGN THE AUDIT PROCESS
BECAUSE IT'S CLEARLY A BURDEN
EVERYONE ON THE CALL HAS BEEN
THROUGH THIS AND WE KNOW WHAT
TIME IT TAKES AND KNOW IT REALLY
IS A DIFFICULT ISSUE AND SO
BASICALLY WE THOUGHT A PAST
ANALYSIS OF PAST RESULTS WOULD
BE THE FIRST STEP AND I THE
RECOMMENDATION WAS ON PROCESS OF
DOING AUDIT TO SEE THERE'S
SOMETHING WE CAN DO TO REDUCE
THE BURDEN.
IT MAY NOT COME AS A SURPRISE TO
EVERYONE THERE'S LIMITED PUBLIC
DATA IN THIS SPEAR.
VERY FEW PUBLICATIONS BECAUSE
COMPANIES DO THEIR AUDITS FOR
REGULATORY PURPOSES AND THEIR
CONFIDENT SLAL INFORMATION.
-- CONFIDENTIAL INFORMATION AND
THEY'RE NOT AVAILABLE TO THE
PUBLIC.
WE DECIDED TO HAVE TWO APPROACH.
ONE, LOOK AT THE DATABASE OF
DEFICIENCY REPORTS THAT EXIST IN
THE CTMB MONITORING BRANCH AND
GO RIGHT TO THE SOURCE IN TERMS
OF THE UA AND REGULATORY STAFF
OF THE COOPERATIVE GROUPS TO SEE
IF THEY HAD IDEAS HOW THE
PROFESSION COULD BE IMPROVED IN
THE AREA WE OVERSEE.
THIS FOCUSSED ON DEFICIENCY
REPORTS FOR THREE YEARS, '22,
'23 AND '24 AND CTMB AND TRIALS.
NOW, FOR THOSE WHO DON'T
REMEMBER BECAUSE IF IF YOU
HAVEN'T HAD THE PLEASURE OF
UNDERGOING ONE OF THESE AUDITS
RECENTLY THEY'RE FOCUSSED ON
THREE BROAD CATEGORIES.
REVIEW OF THE CASES, PHARMACY
REVIEW, WHAT YOU'VE DONE WITH
YOUR IV DRUGS AND REGULATORY
REVIEW.
ONE THING THAT MAY NOT BE
SOMETHING YOU ALL REMEMBER VERY
CLEARLY IS THESE DEFICIENCIES
ARE CLASSIFIED UNDER A WIDE
VARIETY OF CAT GEGOCATEGORIES.
IT'S A VERY COMPLEX BUSINESS.
THE OTHER THING IS THE DATA ARE
NOT COLLECTIVE FOR THE PURPOSE
OF UNDERSTAND THIG AUDIT
PROCESS.
I'LL GIVE YOU MY CONCLUSION NOW
TO SAY THAT THE PROCESS WORKS
WELL AND IN TERMS OF AGGREGATE
DATA IT'S VERY DIFFICULT BECAUSE
THERE'S SO MANY DIFFERENT WAYS
THE DATA ARE CLASSIFIED ARD THE
RULES SAY DEFICIENCIES CAN BE
SINGLE OR IN MULTIPLE ERRORS AND
THE MOST IMPORTANT INFORMATION
WE COLLECT WERE A SINGLE SITE OR
ONE OR TWO TRIALS IS ALL LOCATED
IN THE UNSTRUCTURED DATA
COMMENTS FIELD.
TO GIVE AN UNDERSTANDING OF THE
TABLE BELOW ARE EXAMPLES OF DATA
IN OPEN END DEFICIENCY DATA
FIELD AND RUN AN INCREDIBLE
GAMUT FROM THINGS COUNTED AS
DEFICIENCIES AND THE PATIENT ZIP
CODE AND IF THE TREATMENT WAS
REPORTED INCORRECTLY IS CRITICAL
OR WERE NOT APPROPRIATE HI
CATEGORIZED.
THERE'S A WIDE VARIETY OF
INFORMATION THAT CAN'T BE
QUANTITATED IN THE AGGREGATED
AND IF YOU'RE SITTING WITH AN
AUDITOR AS I HAVE AND GOING OVER
A TRIAL AND PATIENTS MANY ISSUES
ARE VERY IMPORTANT AND THEY ARE
CONTRIBUTE TO THE WAY TRIALS ARE
CONDUCTED AND HOW WELL THEY'RE
CONDUCTED AT PARTICULAR SITES.
SO BASICALLY THE HIGH-LEVEL DATA
IS NOT GIVEN US A LOT OF HELP IN
UNDERSTANDING HOW TO BASICALLY
IMPROVE THE BURDEN.
ONE THING WE DID FIND OUT IS
THAT MAYBE THIS IS OBVIOUS TO
LEARN, MAYBE NOT, BUT IND-EXEMPT
STUDIES HAVE FEWER EFFICIENCIES
THAN IMD TRIALS.
WHAT'S THE REASON?
IS IT BECAUSE THE COMPLEXITY OF
THE TRIALS OR MORE COMPLEX AND
UNDER SCRUTINY.
WHAT'S DIFFICULT GOING FROM THE
DATA FROM THE DATA IS TO
DETERMINE THE RECOMMENDATION
FROM THE PLANNING GROUP WOULD
REQUIRE REVIEW TO GET MORE
SPECIFIC DATA RELATED TO PROCESS
AND HOW TO REDUCE THE BURDEN.
WE HAVE FRUITFUL DISCUSSIONS
WITH OUR STAFF.
THEY'RE THE PEOPLE THAT DEAL
WITH THE NCI AUDITS.
THESE WERE OPEN ENDED QUESTIONS
AND GIVE THE RECOMMENDATIONS THE
GROUP OF INDIVIDUALS CAME UP
WITH.
SO ONE THING IMPORTANT TO POINT
OUT AS WE ALL THINK ABOUT
AUDITS, IT'S SOMETIMES DIFFICULT
TO PARS OUT THE NCTN AUDITS THAN
THE INDUSTRY AUDITS AND THEY'RE
MORE PAINFUL THAN THE NCTN
AUDITS AND IF YOU'RE IN A GROUP
AND PREPARING FOR THESE THINGS
IT'S HARDER FOR THE STAFF TO
KEEP ALL THE DETAILS IN TERMS OF
WHAT WAS INDUSTRY-RELATED BURDEN
TO NCTN-BURDEN CLEAR.
THE PREPARATION FOR THE AUDITS
IS A SIGNIFICANT BURDEN AND
TAKES DAYS AND DAYS.
I'LL SHARE IN MY PREVIOUS EMPLOY
WE HAD A CONFERENCE ROOM
DEDICATED AUDITS BECAUSE WE HAD
SO MANY COMING SO OFTEN.
ALSO WHAT WE GOT FROM THE GROUP
STAFF WAS THAT THERE'S A LOT
VARIABILITY IN LESSER VERSUS
MAJOR IN TERMS OF DEFICIENCY.
THE SYSTEM IS BURDENSOME AND
TRAINING IS DIFFICULT BECAUSE
IT'S CRITICALLY IMPORTANT A LOT
OF REALLY EXPERIENCES PEOPLE
WERE LOST DURING THE PANDEMIC.
WE HAVE TO HAVE THE KIND OF
STAFF TO RESPOND TO THE
PREPARATION AND THE AUDIT
CONDUCT ITSELF.
WE CAN'T HAVE ACTIVITIES THAT
INVOLVE SURGERY AND RADIATION
AND SO THERE HAS TO BE SOME
FLEXIBILITY HOW IT WOULD OCCUR
BUT THERE WAS ENTHUSIASM IN HOW
TO DO SO.
SO WHAT ARE WE GOING TO DO?
I THINK WE HAVE TO CONTINUE TO
EXPLORE WAYS TO STREAMLINE THE
PROCESS.
SO THE FIRST THING THAT IS
OBVIOUS IS THE MORE WE CAN DO TO
DECREASE THE AMOUNT OF DATA THAT
WE COLLECT, THE LESS THE BURDEN
FOR THE AUDIT.
SO THAT REALLY PUTS US AT
GREATER PARITY AT OUR EFFORTS TO
STREAMLINE THE DATA COLLECTION.
THIS HAS BEEN THE HOLY GRAIL IN
TERMS OF DIRECT TRANSMISSION
FROM EHRs TO DATABASES.
A VERY HEAVY LIFT.
A VERY HEAVY LIFT GOING ON FOR
DECADES.
I WOULD LOVE TO BE ABLE TO DO
THAT.
IT COULD DECREASE THE ERRORS I'M
SURE.
THE MONITORING GROUP WOULD LOVE
FEEDBACK AND ONE THING THEY'RE
DOING IN THE AUDITING GUIDELINES
IS TO HAVE A SEPARATE CATEGORY
OR DEFICIENCIES RELATED TO
STUDIES.
15 YEARS AGO WE DID FEWER
CORRELATIVE STUDIES NOW THERE'S
MORE AND MISSING A BLOOD DRAW
FOR A CTNA TEST IS NOT IN THE
SAME CATEGORY AS NOT APPROPRIATE
FOR CATEGORIZING AEs OR
REGULATORY DEFICIENCIES.
HAVING THEM AS A SEPARATE
CATEGORY IS VERY IMPORTANT AND I
THINK WILL DECREASE THE AMOUNT
OF INFORMATION WE NEED TO
RESPOND TO NOT THAT IT'S NOT
IMPORTANT TO GET THE DATA
COLLECTED AND SAMPLES COLLECTED
BUT HAVE TO BE VIEWED AS AN
APPROPRIATE FOR WHAT THE STUDY
IS GOING TO BE SET OUT TO DO AND
WHAT THE QUALITY INSURANCE
ISSUES REALLY ARE.
AND I THINK ANYTHING WE CAN DO
TO WORK WITH THE GROUPS TO
FURTHER HARMONIZE THE PROCESS
ACROSS THE GROUPS AND AUDITORS
WE'D LOVE TO BE ABLE TO DO
WHEREVER THAT'S POSSIBLE.
I THINK SOME OF THE CALL WOULD
LOVE THE NOTION OF DEVELOPING
LIKE FOR THE CENTRAL IRB AND
OTHER SERVICES TO HAVE A
CENTRALIZED PROCESS TO DO ALL
THE AUDITING WOULD BE GREAT.
IT ONLY MAKES IT DIFFICULT TO
DIFFICULT IN THE CURRENT
ADMINISTRATION THOUGH IT WOULD
BE SOMETHING I'D LOVE TO
ENTERTAIN.
I'D LIKE TO THANK ALL THE FOLKS
WHO HELPED INITIATE THE
RECOMMENDATIONS AND THE STAFF
AND CTAC MEMBERS AND CCCT AND
ALL WHO PLAYED A CRITICAL ROLE
IN PUTTING THE INFORMATION
TOGETHER FOR THE ANALYSIS.
HAPPY TO TAKE QUESTIONS.
>> I KNOW IT'S A LOT OF WORK FOR
EVERYBODY BUT IMPORTANT WORK.
QUESTIONS FROM ANYBODY?
>> IT'S TWO SIDES OF THE COIN IN
THE STRUCTURE IT'S ACTUALLY A
LEARNING PROCESS AND A HEAVILY
VOLUNTEERED PROCESS.
WHEN WE PUT TOGETHER AN AUDIT
GROUP OR TEAM YOU'RE EDUCATING
PEOPLE HOW TO MAKE THEIR SITE
BETTER.
THEN THEY DO AUDIT AND THAT
DOESN'T COST THE NCI ANYTHING.
THAT'S VOLUNTEER HOURS.
I DON'T KNOW WHERE ON THE ONE
HAND I WOULD LOVE TO SAY I DON'T
HAVE TO WORRY ABOUT THE AUDIT
PROCESS ANYMORE AS A COOPERATE
GROUP AND THERE'S A CENTRALIZED
PROCESS FOR IT.
ON THE OTHER HAND I THINK IT
HELPS THE COOPERATIVE GROUPS
TRAIN THE NEXT GENERATION AND
HAVE HIGH QUALITY DATA.
ALSO OPPOSED TO THE MORE
CHALLENGING AUDITS FROM FDA OR
INDUSTRY, THE COOPERATIVE GROUPS
IS TRULY AN AUDIT TO TRY TO HELP
PEOPLE IMPROVE THEIR PROCESSES
AND ALWAYS VIEWED AS A POSITIVE
THOUGH THERE'S CHALLENGES
GETTING IT TOGETHER ON THE
INVESTIGATOR END.
YES, IT'S A BURDEN BUT I THINK
WE HAVE TO LOOK AT THE BIG
PICTURES AS WELL.
>> GREAT.
SO WE DON'T NEED TO TAKE A LOT
OF BIG LEEMZ LEAPS FORWARD --
BIG LEAPS FORWARD BUT I WAS
STRUCK FROM COMMENTS FROM THE QA
TEAMS WHERE THEY THOUGHT CHANGES
COULD HELP THE PROCESS.
ONE THAT -- TWO STRUCK ME IN THE
SLIDE.
ONE WAS THE TRAINING.
SO ONE IS TO HARMONIZE THE AUDIT
MANUAL BUT IT'S NOT THE AUDIT
MANUAL ITSELF.
IT'S THE TRAINING THAT GOES HOW
TO USE THE AUDIT MANUAL.
SO MAYBE SOME EFFORT OF GETTING
THE GROUPS TOGETHER TO COME
TOGETHER TO DEVELOP A TRAINING
PROGRAM THAT'S HARMONIZED MAY BE
A SMALL STEP THAT COULD MOVE US
IN THE RIGHT DIRECTION INSTEAD
OF CENTRALIZING EVERYTHING
GETTING THE GROUPS TO COME UP
WITH A COMMON TRAINING PROGRAM
THAT APPLIES TO EVERYBODY.
THAT COULD BE A SMALL STEP THAT
POTENTIALLY COULD HAVE A BIG
IMPACT.
THE OTHER ONE THAT STRUCK ME WAS
THE ISSUE OF AUDIT INFORMATION
SYSTEM.
THERE MAY BE EFFORTS THERE TOO
TO ALLEVIATE THE BURDEN BY
MAKING THAT SYSTEM MORE USER
FRIENDLY, LESS BURDENSOME IN
TERMS OF DETAILS.
THERE MAY BE TWO SMALL STEPS
THAT INCREDIBLY COULD GET US
WHERE WE WANT WITHOUT HAVING TO
CENTRALIZE EVERYTHING.
JUST SOME SUGGESTIONS.
>> THANK YOU.
>> YOU HAVE PROVEN IT'S
DISPARATE AND HARD TO AGGREGATE
AND BUILD A BETTER MORE
SIMPLIFIED SYSTEM AND TELL THEM
WHAT TO DO.
DON'T COLLECT BAD ZIP CODES.
WHO CARES IT WOULD LEAD TO LESS
RESOURCES.
COULD WE PILOT AT 10 SITES A
SIMPLER AUDITING SYSTEM AND
ENSURE IT'S STILL SAFE?
>> CHUCK, I THINK THAT'S
SOMETHING THE GROUPS WANT TO DO,
WE NEED TO START THINKING ABOUT
IT.
>> CAROLINE.
>> I AGREE IN A BALANCED WAY
WITH WHAT ROBERT SAID.
WE TAKE THEM AS EDUCATIONAL
OPPORTUNITIES BUT WE HAVE TO
REMEMBER WHY WE'RE DOING AUDITS
FOR INTEGRITY OF THE TRIAL AND
NOT PATIENT SAFETY.
EVERYBODY WANTS TO DO ATTENTION
TO DETAIL BUT WE'RE HUMAN AND
THE OTHER ASPECT OF ALL OF THE
AUDITING PREP IS WE PUT CAP IN
PLACE TO ADDRESS THE ISSUES THAT
WE DISCOVER BUT IF WE DON'T HAVE
THE AVAILABILITY TO MONITOR
BECAUSE WE'RE ALWAYS IN AUDIT
PROCESS, WE DON'T KNOW WE'RE
SUCCESSFULLY ADAPTING OUR CAP
AND EVERYTHING LIKE THAT.
IT'S A BALANCE OF THE BURDEN AND
WHEN THE AUDITORS PULL CHARTS
FROM THREE YEARS AGO AND WE
DISCOVERED THE PROBLEMS AND MADE
A CAP BUT THE NEXT AUDIT IS
STILL SEEING THE BACK TIMING
THEN WE'RE JUST REHASHING THE
SAME THING.
THERE'S GOT TO BE A BALANCE WITH
ALL OF THAT.
>> ABOUT POINT, EVERYBODY, MADE.
TWO COMMENTS.
FIRST, I AGREE THERE'S AN
EDUCATIONAL COMPONENT TO ALL
THIS GIVEN THE PANDEMIC AND
ANTICIPATED LOSS OF STAFF AND SO
ON BUT I AGREE WITH CARE LINE
THAT WE CAN'T LOSE SIGHT OF
THAT.
FALLING ON THAT I WONDER IF
THERE'S A WAY TO HAVE THE DATA
ELEMENTS AUDITED FOR INSTANCE
THE ZIP CODE IS NOT ON ITS FACE
WOULD AFFECT PATIENT SAFETY AND
COULD THOSE BE PRIORITIZED IN A
RISK-BASED APPROACH.
THAT'S THE FIRST COMMENT.
THE SECOND IS THAT THE PROCESS
IS ONEROUS AND IT'S THE
FOLLOW-UP THAT TAKES PLACE IS
OFTEN MORE ONEROUS AND THE
AUDITOR COMES AND THE UNSPOKEN
THREAT IS WE'RE GOING TO PUT YOU
ON ENROLLMENT HOLD OR SOMETHING
BECAUSE WE DISCOVERED SOME MAJOR
THING AND EVERYBODY GETS
NERVOUS.
FOR THE NCI IS THERE A WAY THE
CONSEQUENCES COULD NOT BE MADE
LESS MEANINGFUL BUT MADE MORE
TRANSPARENT SO IS THE SITES HAVE
LESS FEAR OF THE CONSEQUENCES OR
HAVE MORE INPUT OR CONTROL TO
HOW THEY'RE MANAGED.
THAT MAY HELP ALLEVIATE THE
ANXIETY AROUND THE ACTUAL AUDIT
PROCESS ITSELF WHICH THEN FEEDS
INTO HOW MUCH TIME PEOPLE PUT
INTO PREPARING FOR IT.
THOSE ARE TWO COMMENTS.
>> THERE'S A LOT OF VARIABILITY
AND HARMONIZE SITES WOULD BE
HELPFUL.
WE NEED TO MOVE ON AND ANDREA
DENICOFF WILL TALK ABOUT THE
SITES MORE.
>> THANK YOU FOR THE OPPORTUNITY
TO SHARE THIS WORK WITH YOU.
THIS IS DECENTRALIZED CLINICAL
TRIAL ELEMENTS AND DESCRIBE HOW
WE'RE IMPLEMENTING IT.
AS MANY ARE AWARE, MANY
DECENTRALIZED ELEMENTS WITHIN A
TRIAL WERE FOUND TO BE EXTREMELY
BENEFICIAL TO PATIENTS DURING
THE COVID-19 PANDEMIC AND AS WE
WERE COMING OUT OF THE PANDEMIC
EVERYONE REACHED OUT TO US AND
SAID WE WANT TO CONTINUE MANY OF
THESE DECENTRALIZED ACTIVITIES
AS THEY'RE HELPFUL TO PATIENTS
AND THE WAS CREATING GUIDANCE ON
ACTIVITIES AND IN PARALLEL THE
RESEARCH BASES REALLY WANTED TO
WORK TOGETHER WITH CTAC AND DCP
TO SEE IF WE CAN CREATE COMMONLY
USED LANGUAGE SO ALL COULD
UNDERSTAND WITHIN THE TRIAL.
WHAT THE CTAC RESEARCH BASES
THOUGHT WOULD BE HELPFUL WAS TO
CREATE A CHECKLIST TO HELP
PROTOCOL AUTHORIZE AND STUDY
TEAMS TO LOOK AT WHAT COULD BE
DECENTRALIZED AND -- THE DCT,
COULD CREATE LANGUAGE TO BE USED
ACROSS THE PROTOCOLS SO SITES
AND HAVE A BETTER UNDERSTANDING.
THAT'S WHAT OUR NCTN/NCORP TEAM
DID CIRCULATING SOME LANGUAGE.
FOR THOSE COMMONLY DE SEN IZED
AND TO HAVE THE OPPORTUNITY FOR
THE WORK WOULD BE REMOTE CONSENT
AND NOT A REGISTERED
INVESTIGATOR.
TELEHEALTH VISITS WITH STUDY
TEAM FORMING LOCAL LABS, AND
SINCE THE START OF THE PANDEMIC
WE WERE SHIPPING AGENTS DIRECTLY
TO PARTICIPANTS AND WHERE
POSSIBLE TREATMENTS FOR
INVESTIGATIONAL THERAPIES FOR A
TRIAL AND IF POSSIBLE RADIATION
THERAPY DONE MORE CLOSE TO A
PATIENT'S HOME.
AT THE BOTTOM IS DIFFERENT
SCENARIOS FOR SCENARIOS IN THE
CHECKLIST WE HEARD BACK FROM ALL
THE GROUPS THAT IS HELPFUL TO
PROMPT STUDY TEAMS FOR
ACTIVITIES THAT COULD BE
DECENTRALIZED WITHIN A TRIAL.
WE HAVE ONE TRIAL CURRENTLY
ACTIVE, A PHASE III STUDY FOR
PATIENTS WITH METASTATIC
PROSTATE CANCER AND IT'S AN
IND-EXEMPT TRIAL SO IT HAS
SEVERAL COMMERCIAL AGENTS AND
ONE INCLUDES THE ADMINISTRATION
OF COMMERCIAL AGENTS COULD BE
DONE BY A LOCAL ONCOLOGIST AND
ELEMENTS WITH THE STUDY TEAM
SUCH AS THOSE IN ASSESSING
ADVERSE EVENTS AND CAN HAVE
THEIR LOCAL LAB TESTS AND IN
PARTICULAR IS FOR
PROGRESSION-FREE SURVIVAL AND IN
THIS CASE THE PATIENT COULD NOT
HAVE IMAGING STUDIES DONE
LOCALLY.
THEY NEED TO COME TO THE TRIAL
SITE BECAUSE OF THE END POINT
AND DEPENDING ON THE TRIAL
CENTRALIZED ACTIVITIES WORK
BETTER THAN OTHERS DEPENDING ON
THE DESIGN OF THE TRIAL.
>> ONE OF THE ISSUES WAS THE
DEBATE BETWEEN THE RESPONSIBLE
INVESTIGATOR AND LOCAL HEALTH
CARE PROVIDER OR NON-REGISTERED
INVESTIGATOR.
THROUGH DISCUSSION WITH THE
GROUPS AND CENTRALIZED IRB
DISCUSSED THE OVERSIGHT BY THE
RESPONSIBLE INVESTIGATOR OR THE
ENROLLING SITE INVESTIGATOR
WOULD HAVE TO HAVE AN AGREEMENT
WITH THAT OF THE LOCAL
ONCOLOGIST PROVIDER WITH THE
STANDARD OF CARE WITH COMMERCIAL
AGENTS BECAUSE WE NEED TO HAVE
THE DATA RECORD AS PART OF THE
TRIAL SO THAT FOR STUDIES UNDER
THE NCIB PART OF THE INSTITUTION
WORK SHEET FOR THE NCIB THEY
PRODUCED IT THEY DO EVERY YEAR
THEY HAD TO INCLUDE THE
AGREEMENT WITH THE LOCAL HEALTH
CARE PROVIDERS BECAUSE MANY
SITES USE THE SAME LOCAL HEALTH
CARE PROVIDERS WITHIN THEIR SITE
AND THIS SHOULD BE PART OF THE
APPROACH FOR CAPTURING STUDY
RECORDS.
WE CLEARED THE WAY FOR THIS TO
MAKE IT POSSIBLE FOR THIS TO
HAPPEN WITH THE AGREEMENT.
SOME OF THE TRIAL ELEMENTS NOT
DECENTRALIZED IN THE TRIALS FOR
THE INVESTIGATIONAL AGENTS
DELIVERED VIA IV HAS THE
POTENTIAL FOR IT BUT WOULD NEED
FDA REVIEW AND APPROVAL.
THE CURRENT FDA GUIDANCE I SITED
ON THE EARLIER SLIDE HAS A
STATEMENT THAT TALKS ABOUT
TRIALS WHERE ALL ACTIVITIES
WHERE DECENTRALIZED MAY BE
APPROPRIATE FOR INVESTIGATIONAL
PRODUCTS WITH WELL CHARACTERIZED
SAFETY PROFILES AND DO NOT
REQUIRE ADMINISTRATION OR
MEDICAL ASSESSMENTS.
IT'S STILL POTENTIAL OPTION JUST
FOR THE FDA REVIEW AND APPROVAL
AND CERTAINLY WORTH CONSIDERING
IN SOME TRIALS.
THAT'S IT FOR THE DECENTRALIZED
TRIAL ACTIVITIES.
SHOULD WE DO COMMENTS OR
QUESTIONS NOW OR GO ON TO THE
NEXT --
>> GO AHEAD AND DO THE NEXT
TOPIC AND THEN TAKE THEM ALL AT
ONCE.
>> OKAY.
SOUNDS GREAT.
THE NEXT TOPIC IS TE
STREAMLINING OF DATA COLLECTION
OR IMD EXEMPT TRIALS AND I'M
PRESENTING THIS ON BEHALF OF MY
CO-CHAIR OF THE STREAMLINE
IMPLEMENTATION COMMITTEE AND I'M
PRESENTING ON WE HALF OF BOTH OF
US TODAY.
WE UNDERSTAND THE TERRIFIC AND
GREAT NEED TO STREAMLINE OUR
CLINICAL TRIALS DATA AND THE
CLINICAL TRIALS WORKING GROUP
CAME UP WITH THE IDEA OF COMING
UP WITH STANDARD PRACTICES FOR
STREAMLINING DATA AND REDUCE THE
OPERATIONAL BURDEN OF THE TRIALS
AND THE INITIAL
[NO AUDIO]
AND WERE IND-EXEMPT AND WHEN WE
LOOKED AT THE PORTFOLIO TO
REPRESENT ABOUT 30%-35% OF OUR
TRIALS SO IT WOULD BE A GOOD
PROPORTION TO HAVE REDUCED DATA
TRIALS FOR THE SITES.
I THINK EVERYONE CAN AGREE THE
BIGGEST BURDEN IS COLLECTING
ADVERSE EVENT DATA.
THE STREAMLINING STANDARD
PRACTICE FOR THIS DATA ELEMENT
WOULD BE THAT FOR THESE IND
EXEMPT TRIALS WOULD ONLY COLLECT
GRADE 3 ADVERSE EVENTS OR HIGHER
UNLESS THERE'S SOME SPECIFIC
STATED OBJECTIVE WHY YOU NEED TO
COLLECT A LOWER GRADE AE IN
ADDITION TO ONLY COLLECTING
GRADE 3 OR HIGHER, THE ELEMENTS
WOULD NOT HAVE THE ATTRIBUTION
OF DATA COLLECTED AND WOULD
SIMPLIFY AND STREAMLINE THIS
MORE AND ADVERSE DATA HAS BEEN
RESEARCHED IN THE PAST AND
THINGS LIKE AE ATTRIBUTION HAVE
NOT BEEN USED IN MOST
PUBLICATIONS WHEN DESCRIBING THE
ADVERSE EVENTS FOR DATA USED IN
THE STUDY.
SO THE OTHER DATA CATEGORIES
WERE ALSO LOOKED AT TO BE
STREAMLINED AS PART OF THE
STANDARD PRACTICES AND THE DATA
IN THE CATEGORIES SHOULD ONLY BE
SUBMITTED IF THERE'S SOME
SPECIFIC NEED FOR END POINT
DATA, STRATIFICATION FACTORS, IF
IT'S A PATIENT CHARACTERISTIC
NEEDED TO DESCRIBE THE PATIENT
POPULATION OR PUBLICATION FOR
DETERMINING ELIGIBILITY OF
TREATMENT.
SO THIS IS SOMETHING THAT EACH
OF GROUPS HAD TO DO INDIVIDUALLY
BECAUSE EACH GROUP HAS THEIR OWN
CASE REPORT FORM THEY UTILIZED.
SO EACH GROUP REALLY HAD TO DO A
LOT OF WORK IN STREAMLINING
THESE ELEMENTS FOR IND-EXEMPT
STUDIES.
SO THE STREAMLINE DATA PRACTICES
WERE IMPLEMENTED WE STARTED
IMPLEMENTING THEM JANUARY 1,
2025.
AND THE ENTIRE GROUP DECIDED THE
STANDARD PRACTICE WOULD FIRST
HAVE TO REQUIRE AN APPROVAL
WITHIN THEIR OWN GROUP FIRST.
AND REQUIRE APPROVAL AND IF THAT
WAS APPROVED THEN SUBMITTED TO
CTAC AND THEN CTAC DURING
PROTOCOL REVIEW COULD DECIDE IF
THERE WAS A COMPELLING REASON TO
HAVE THE EXPANDED DATA
COLLECTION NEEDED OR WAS IT
JUSTIFIED FOR SCIENTIFIC
RATIONALE.
AND WORKING WITH THE CHAIRS OF
THIS COMMITTEE WE WERE MEETING
QUARTERLY TO SEE HOW THE
IMPLEMENTATION IS GOING.
WE'RE -- THE GROUP ITSELF ARE
REALLY A TERRIFIC GROUP.
THEY'RE WORKING TOGETHER TO
SHARE SITE STAFF EDUCATION.
WORKING WITH EDUCATING THEIR
INVESTIGATOR COMMUNITY.
AND GROUPS WERE TALKING ABOUT
HARMONIZING IN A MORE STREAMLINE
FASHION THE REPORTING CRITERIA
AND MAYBE HAVING AN FAQ DOCUMENT
TO CAPTURE LESSONS LEARNED TO
SHARE WITH EACH OTHER BECAUSE WE
HAD GROUPS HAVE A FEW STUDIES
AND ONE OR TWO GROUPS THAT
HAVEN'T HAD AN IND EXEMPT STUDY
SO FAR.
SO HERE'S WHERE WE ARE TO DATE
SO FAR.
WE'VE ALREADY ACTIVATED NINE
PHASE III TRIALS WITH STREAMLINE
ACTIVATION AND THREE UNDER
REVIEW AND WE HAVEN'T HAD A
SINGLE GROUP REQUEST AN
EXCEPTION FROM THE STREAMLINE
DATA COLLECTION FOR THE TRIALS
SO FAR SO I HAVE TO REALLY SING
THE PRAISES OF ALL THE GROUPS TO
EMBRACE THIS.
I THINK ON MY NEXT SLIDE I LIST
WHOSE PART OF THE TEAM.
NOT YET.
IT'S COMING.
SO NEXT STEPS.
WHAT ARE WITH DOING NEXT?
SO AROUND WE HAD GROUPS
INTERESTED IN DOING THE NCOR
STUDIES AND WANT TO EXPLORE IS
THERE AA WAY TO REDUCE -- --
THERE'S A WAY TO REDUCE ISSUES
AND NEED A SPECIALIZED THOUGHT
ABOUT THOSE SPECIFIC ELEMENTS.
AND FUTURE CONSIDERATIONS FOR
CTAC.
HIND STUDIES ARE FEASIBLE TO USE
THE DATA COLLECTION STANDARDS.
AND WHILE WE'RE DOING THIS IN
ABOUT 30% OF OUR TRIALS, AND
DATA COLLECTION BURDEN SO THERE
ARE WAYS WE CAN STREAMLINE
COLLECTION IN IND STUDIES.
AND LOOK AT A PARTNERSHIP AND
MORE OF THAT EXAMPLE ROLLOUT FOR
OTHER IND TRIALS.
AND SHOULD CTAC CONSIDER FORMING
THE WORKING GROUP AND CONSIDER
STRATEGIES FOR ELIMINATING DATA
COLLECTION FOR IND STUDIES
WITHIN ALL THE APPROPRIATE
CONTEXTS.
AND I THINK THE FEEDBACK WE'RE
GETTING ON OUR QUARTERLY CALLSES
ELEMENT AT LEAST THE GROUPS THAT
SUCCESSFULLY LAUNCHED THE TRIALS
OUT THERE IS THAT WHEN THEY
BROUGHT THIS BACK TO THE
PROTOCOL WRITING TEAM AND THEIR
DATA DEVELOPMENT TEAM IS THAT
THEY'RE EMBRACING THIS.
SOME OF THIS THERE'S A QUESTION
ABOUT CAN I STILL ASK THIS
QUESTION AND THIS QUESTION BUT
WE'RE REALLY TRYING TO MAKE IT
SO THAT THE DATA ELEMENTS IN A
TRIAL HAVE A SCIENTIFIC MERIT TO
THEM ASKING THEM AND NOT THAT WE
MIGHT DO THIS AND THAT AND THIS
PHASE III IND-EXEMPT TRIAL WE
LIKE TO FOCUS ON THE COMPELLING
SCIENCE AND I'LL STOP THERE AND
TAKE ANY QUESTIONS.
>> DO YOU HAVE QUESTIONS?
>> AND OFTEN A CLINICAL SITE
DOESN'T HAVE RESOURCES TO HELP
ADDRESS THE PATIENT'S NEEDS
WHEREAS THEY MIGHT HAVE THEM AT
HOME AND THAT RELIEVES THE
SUPPORT PERSON OF THE BURDEN FOR
THE HOSPITAL AND SO I THINK
THAT'S REALLY COOL ADVANTAGE FOR
REMOTE CONSENT AND ENCOURAGE
THEM TO USE THAT.
>> GREAT POINT.
I TOTALLY AGREE.
>> THANK YOU SO MUCH.
PATTY?
>> HI, ANDREA.
BOTH OF THESE TOPICS ARE VERY
IMPORTANT TO ME AS YOU KNOW.
THAT WILL DECREASE DEVIATIONS AS
WELL AS THE AUDIT PROCESS AND IT
SAYS DECENTRALIZED IN THE STUDY
AND TITLE BUT THEN YOU DON'T
READ IT ANYWHERE AND NOW WE'RE
MOVING TO THE AND CONSENT FORM
LANGUAGE AS WELL.
>> YOU KNOW, WE TALKED ABOUT
THAT AND PEOPLE DIDN'T THINK IT
NEEDED TO BE IN THE CONSENT FORM
BECAUSE THE CONSENT DOESN'T GET
DOWN TO THE GRANULAR OF WHERE
THE ACTUAL ACTIVITIES TAKE
PLACE.
AND SOME SITES ARE MORE AMENABLE
TO DOING THEM THAN OTHERS AND
THOSE ARE NATIONAL CONSENT.
AND LOOK AT THE CENTRALIZED
TRIALS AND SOME CALL IT
DECENTRALIZED ACTIVITIES AND
SOME CALL IT CONTINUITY OF CARE.
IT'S IN THE APPENDIX.
>> THANK YOU.
>> AND LOOK AT THE LOCAL
PROCESSES AND REPORTING OF AEs.
I THINK MANY OF US ARE STILL
STUCK IN THIS MODE OF HAVING TO
CONTINUE TO ATTRIBUTE FOR EVERY
SINGLE THING THAT OCCURS TO A
PATIENT ON AN NCTN TRIAL AND I
WOULD JUST ENCOURAGE US TO
FIGURE OUT HOW WE GET THAT
MESSAGE TO THE CANCER CENTERS
AND HELP THEM GET UP TO SPEED
WITH THIS INITIATIVE.
WE WERE A TEST CASE WITH 2312
AND I'M EXCITED TO BE ABLE TO DO
THIS.
HOW DO WE KNOW IF IT'S WORKING?
ARE THERE -- IS THERE A PROCESS
IN PLACE TO SAY OKAY, THIS IS
WORKING, THIS ISN'T WORKING?
SOME SUBJECTIVE EVALUATION?
HAVE YOU THOUGHT ABOUT THAT?
I'M SURE YOU'VE THOUGHT ABOUT
THAT.
>> WE HAVE THOUGHT ABOUT IT.
WE DIDN'T WANT TO ADD ANOTHER
BURDEN TO THE SITES OF MORE DATA
BEING COLLECTED BUT THOUGHT WE'D
DO IT MORE BY GOING TO THE NCTN
GROUP MEETINGS AND HAVING DIRECT
DIALOGUE WITH THE SITES.
THEY ALL HAVE SITE OPERATION
COMMITTEES AND WE PRESENTED THE
TOPIC.
WE'LL HAVE TO DO -- WE DIDN'T
FEEL THERE WAS A NEED FOR FORMAL
DATA COLLECTION IN TERMS OF THE
DECENTRALIZED ACTIVITY.
>> WE NEED TO MOVE THE LIMITED
DATA TO THE IND STUDIES.
THAT WAS ALL THE GOAL.
I THINK WE'VE SHOWN THE PROCESS
WORKS.
WE NEED TO TAKE THE NEXT STEP.
I'M NOT SAYING REGISTRY STUDIES
AT THIS POINT BUT CERTAINLY IND
STUDIES THAT AND WOULD BE REALLY
HELPFUL WITH THE AUDITING
BURDEN.
>> IT WOULD HELP EVERYTHING.
>> JACK.
>> THOUGH THIS IS A NO-BRAINER
IT TAKES A SURPRISING AMOUNT OF
EDUCATION OF YOUR MEMBERS OF
YOUR NETWORK OR CANCER CENTER
NETWORK OR GROUP NETWORK.
I CAN'T TELL YOU HOW MANY TIMES
PEOPLE COME BACK LATER AND SAY I
WAS GOING TO DO A PAPER ON THE
EFFECT OF SMOKING WITH THIS
DRUG.
YOU'RE NOT GOING TO BE DOING
THIS IS REQUIRED.
>> ALL RIGHT.
>> THANK YOU SO MUCH.
GREAT TO SEE ALMOST A DOZEN
TRIALS ON THEIR OWN WITH NEW
REQUIREMENTS.
MY COMMENT RELATES TO THE
DECENTRALIZED TRIAL ELEMENTS.
YOU'VE HAD A GREAT LIST AND EACH
WILL MAKE A DIFFERENCE.
THE ONE I WANTED TO ASK MORE
ABOUT IS THE ADMINISTRATION OF
COMMERCIAL PRODUCTS BY A LOCAL
HCP.
YOU DESCRIBED SOME REQUIREMENTS
AND UNDERSTAND WHAT NEEDS TO
HAPPEN FOR ME TO BE COMFORTABLE
WITH THE LOCAL PHYSICIAN NOT
PART OF MY HEALTH SYSTEM
ADMINISTER THE DRUG.
IF THE PROCESS IS NOT SIMPLE
ENOUGH INVESTIGATORS MAY SHY
AWAY FROM USING THAT MECHANISM.
JUST WANTED TO HEAR YOUR
THOUGHTS AND MAKE IT AS SIMPLE
AS POSSIBLE.
>> YEAH, IT COULD BE AS SIMPLE
AS THE AGREEMENT COULD BE BACK
AND FORTH DOCUMENTATION.
THIS PATIENT IS ON THIS CLINICAL
TRIAL.
THIS IS THE -- IF THERE IS A
SPECIFIED IT DEPENDING ON THE
TRIAL.
SOME TRIALS JUST SAY DON'T
SPECIFY THE DETAILS OF THE
STANDARD OF CARE ARM IT COULD BE
PHYSICIANS CHOICE.
WHATEVER THE PATIENT AND THE
RESPONSIBLE INVESTIGATOR AT SITE
HAVE DETERMINED, YOU MAKE THE
CONNECTION WITH THE LOCAL HEALTH
CARE PROVIDER AND HAVE IT HAVE A
DOCUMENT LIKE SOMETHING LIKE
WITH E-MAIL AND YOUR SITE NEEDS
TO HAVE AS PART OF ITS ANNUAL
SIGNATORY WORK SHEET WITH THE
IRB YOU HAVE RELATIONSHIPS WITH
LOCAL ONCOLOGISTS TO PROVIDE
STANDARD OF CARE COMMERCIAL
AGENTS.
SO IT REALLY DOESN'T HAVE TO
BE -- WHEN I WAS IN PRACTICE WE
DID THIS A LOT AND IT WASN'T A
BIG DEAL AND WAS TERRIFIC FOR
PATIENTS.
SO SO, HOPEFULLY THAT DOESN'T
SOUND TOO BURDENSOME.
>> ALL RIGHT.
ANY OTHER QUESTIONS FOR ANDREA?
IF NOT THANKS SO MUCH.
WE APPRECIATE THE WORK.
>> ALL RIGHT.
WE'RE GOING TO MOVE ON TO THE
LAST PART THE NEW BUSINESS PART
AND TURN IT OVER TO SHEILA.
>> TERRIFIC.
CAN WE BRING UP THE SLIDES?
GOOD AFTERNOON, EVERYONE.
AND SO GLAD WE WERE ABLE TO
CONVENE TODAY.
AS YOU ALL KNOW, CTAC PLAYS AN
IMPORTANT ROLE IN SHARING THAT
NCI REMAINS RESPONSIVE TO THE
NEEDS OF PATIENTS AS WELL AS THE
RESEARCH COMMUNITY.
THE WORKING GROUPS ARE FORMED
PERIODICALLY UNDER THE AUSPICES
OF CTAC TO LOOK AT TOPICS AND
CHALLENGES FACING THE CLINICAL
TRIALS OPERATION IN RESEARCH.
AND TO COME BACK AND PROVIDE
RECOMMENDATION TO CTAC AND NCI.
ZA TODAY WE'D LIKE TO CONSIDER
THE FORMATION OF THREE WORKING
GROUPS TO HELP US MOVE FORWARD.
PARTICULARLY CONTINUE WORKING
DISCUSSION DURING THE CHALLENGES
TIMES.
AND THE PERSPECTIVES IN CANCER
RESEARCH AND CLINICAL TRIAL
INNOVATIONS WORKING GROUP.
THE FIRST I DON'T THINK I NEED
TO SAY TOO MUCH MORE.
ANDREA HAS SPOKEN TO YOU ABOUT
THE NEED CTAC ALWAYS ENVISIONED
INCORPORATING AND STREAMLINING
IND-EXEMPT TRIALS.
THE PURPOSE OF THE STREAMLINING
DATA COLLECTION IN CLINICAL
TRIALS WORKING GROUP WOULD BE
LIKE VERSION 2.
THE PURPOSE IS TO ADVISE CTAC
AND THE NCI DIRECTOR ON
ADDITIONAL STRATEGIES FOR
LIMITING DATA COLLECTION AND NCI
CLINICAL TRIALS.
AND THESE STRATEGIES MAY INCLUDE
DEVELOPING PRACTICES FOR IND
TRIAL IN THE RIGHT CLINICAL
SCIENTIFIC AND REGULATORY
CONTEXT.
SO THE MEMBERSHIP WILL INCLUDE
REPRESENTS FROM CTAC AND OTHER
INDIVIDUALS WITH RELEVANT
EXPERTISE.
IT'S A CRITICAL INITIATIVE TO
HAVE FDA COLLEAGUES
PARTICIPATING IN AS WELL AS WE
CONSIDER IND STUDIES.
THAT'S IT FOR THE WORKING GROUP.
SO WHAT WE NEED TO DO
PROCEDURALLY IS PAUSE AFTER EACH
OF THESE TOPICS AND TAKE A VOTE
FOR EACH MUCH THE WORKING
GROUPS, OKAY.
I WILL FINISH AND HAVE
DISCUSSION AND THEN TALK ABOUT
THE OTHER TWO GROUPS.
>> OKAY.
ANY DISCUSSION OR QUESTIONS
ABOUT THIS?
>> IT I THINK SINCE IT'S AN IND
IS ONE OF THE PROCESSES HERE ON
THAT WORKING GROUP SHOULD BE
SOME FORM OF INDUSTRY
REPRESENTATION.
>> WE'LL TAKE A VOTE ON THIS.
ANYONE OPPOSED TO HAVING THIS
STREAMLINING DATA COLLECTION
WORKING GROUP?
SEEING NONE, I BELIEVE IT
PASSES.
THANK YOU.
>> OKAY.
THANK YOU.
CAN WE MOVE TO THE NEXT SLIDE?
SECOND IS A WORKING GROUP
FOCUSSED ON PATIENT PERSPECTIVES
ON CANCER RESEARCH.
WE HEARD FROM DR. LOWY EARLIER
NOW MORE THAN EVER IT'S
IMPORTANT TO HAVE A VENUE TO
INFORM OUR RESEARCH EFFORTS.
THE PURPOSE OF THIS WORKING
GROUP WOULD BE TO PROVIDE THE
COLLECTIVE PATIENT PERSPECTIVE
ON CLINICAL AND TRANSLATIONAL
RESEARCH INITIATIVES TO ADVISE
CTAC AND ACTIVITIES THAT FOSTER
MEANINGFUL ENGAGEMENT BETWEEN
NCI AND THE PATIENT COMMUNITY,
PARTICULARLY IN AREAS OF
EMERGING RESEARCH.
AND THEN OF COURSE WE WANT TO
MAKE SURE WE'RE ABLE TO PROVIDE
PATIENT-CENTERED INPUT TO INFORM
OUR RESEARCH PROGRAMS.
SO MEMBERSHIP FOR THIS GROUP
WOULD INCLUDE REPRESENTATIVES
FROM CTAC.
WE HAVE ONE PATIENT ADVOCATE.
WE HAVE A SLOT FOR ANOTHER AND
ENVISION THE CTAC ADVOCATES
WOULD BE ON THE GROUP AS WELL AS
OTHERS FROM THE CANCER COMMUNITY
WITH RELEVANT EXPERTISE.
I DON'T KNOW IF THERE'S ANY
OTHER COMMENTS FROM CTAC
MEMBERS.
YOU CAN OPEN IT UP.
>> ANY DISCUSSION OR COMMENTS?
>> THIS IS A WONDERFUL IDEA.
ONE QUESTION.
IS THE SCOPE FOR THIS GROUP
REALLY PATIENT ADVISORY GROUP
INPUT ON THE BROADER RESEARCH
INITIATIVES OR DOES IT INCLUDE
WITHIN SCOPE HOW WE FACTOR IN
THE PATIENT VOICE IN TERMS OF
THE DESIGN OR CONDUCT OF
INDIVIDUAL TRIALS?
>> IT'S AN OPPORTUNITY FOR THE
PATIENT COMMUNITY TO HEAR ABOUT
OUR RESEARCH ACTIVITIES AND
PROVIDE INPUT BACK TO US AS WELL
AS NEW THINGS ALONG THE LINES OF
WHAT THE PATIENT COMMUNITY VIEWS
AS THE NEED FOR US TO BE AWARE
OF NCI'S RESEARCH PROGRAMS.
I DON'T KNOW IF PATTY OR VINCENT
FROM OUR ADVOCACY RESEARCH
OFFICE ANY ADDITIONAL COMMENTS
YOU WANT TO MAKE TO RUBEN'S
QUESTION.
>> I THINK FOR ME IT'S REALLY BY
DIRECTIONAL.
DURING THE TIMES OF REAL
UNCERTAINTY AND WHAT'S GOING ON
I THINK IT'S REALLY IMPORTANT TO
HAVE SOMETHING LIKE THIS AND
LOOK AT CLINICAL TRIALS AND
THERE'S SO MANY PROGRAMS.
THERE'S A LOT OF ORGANIZATION
WITHIN CTAC WOULD BE NICE TO
HAVE.
AND IT'S ABOUT THE ATMOSPHERE
AND THERE'S PATIENT TRUST AND
THE MORE WE CAN HAVE AT THE
VOICE AND EVERYTHING DONE AT NCI
THE BETTER IT'S GOING TO BE
PERCEIVED BY THE COMMUNITY AND
THE PUBLIC IN IN STILLING MORE
TRUST.
I THINK IT'S IMPORTANT AND THE
TIMING IS IMPORTANT.
>> I WANTED TO MAKE SURE IT'S
CONSIDERED BROADLY NOT JUST
UNDER THERAPY BUT SURVIVORS,
COMMUNITY MEMBERS, INDIVIDUALS
INVOLVED IN PREVENTION AND
SCREENING TRIALS PARTICULARLY AS
MORE OF THOSE ARE BROUGHT
FORWARD I THINK IS REALLY
IMPORTANT.
JUST SIMPLY UNDER LINING WHAT
SHE STATED NEEDS TO BE BROADLY
INCLUSIVE.
THANK YOU.
>> ALL RIGHT.
ANY OTHER COMMENTS FOR THIS
PERSPECTIVE?
ALL RIGHT.
ANYBODY OPPOSED TO THIS AS AN
INITIATIVE?
HEARING NONE, THAT PASSES.
THANK YOU.
MOVING ON TO THE NEXT ONE?
>> WE NEED TO VOTE.
OKAY.
ALL RIGHT.
ANYONE IN FAVOR OF THE
INITIATIVE, PLEASE RAISE YOUR
HAND.
ALL RIGHT.
ANYONE OPPOSED?
I BELIEVE IT PASSES.
THANK YOU.
>> THANK YOU.
WE CAN MOVE TO THE NEXT SLIDE.
THE FINAL ONE WE WOULD GROUP IS
THE CLINICAL TRIAL INNOVATION
WORKING GROUP.
AS MANY RECALL NCI FORMS A
CLINICAL TRIALS INNOVATION UNIT
FOR OPERATIONAL EFFICIENCIES FOR
HIGH PRIORITY RESEARCH NEEDS IN
2023.
CTAC HEARD ABOUT THIS SO I'M
HOPEFUL SOME RECALL THAT ABOUT
THE INITIATIVE EARLY ON BUT
SINCE THAT TIME THEY LAUNCHED A
HIGHLY PRAGMATIC TRIAL KNOWN AS
A PROSPECT LAUNCHED BY ALLIANCE
AND THAT IS AIMING TO
RE-EVALUATE THE ROLE OF
IMMUNOTHERAPY WITH PATIENTS WITH
NON-SMALL CELL LUNG CANCER.
IN THE PRESENT ENVIRONMENT NCI
WOULD LIKE TO BRING IT UNDER THE
AUSPICES OF CTAC BY FORMING A
WORKING GROUP FOR RESEARCH
OPPORTUNITIES AND TO BUILD ON
THE PROGRESS ON THE WORK WITH
THE COMMUNITY PARTICULARLY THE
GROUP CHAIR LEADERSHIP AND FDA
ONCOLOGY OF EXCELLENCE AND NCI.
IT'S BEEN A GOOD FORUM AND WE
ALSO WOULD LIKE TO LOOK AT THE
PROGRESS OF THE WORK THIS GROUP
HAS ALREADY BEGUN.
THE PURPOSE IS TO ADVISE CTAC
AND THE NCI DIRECTOR
TRANSFORMATIVE APPROACHES TO
CLINICAL TRIALS DRAWING ON THE
WORK OF THE CLINICAL TRIALS
INNOVATION UNIT AND STRATEGIES
THAT MAY BE DISCUSSED AND HAVE
FURTHER DISCUSSION INCLUDING
ADVANCING PRAGMATIC TRIALS AND
REAL WORLD DATA AND MEMBERSHIP
INCLUDE REPRESENTATIVES FROM
CTAC AND OTHER INDIVIDUALS WITH
RELEVANT EXPERTISE INCLUDING THE
NCTN LEADERSHIP AND
REPRESENTATIVES NCI AND THANK
YOU I'LL TURN IT BACK TO YOU.
>> ANYBODY WITH DISCUSSION OR
QUESTIONS ABOUT THE TOPIC?
>> OKAY.
ERNIE.
>> THE RECENT ANNOUNCEMENT
REGARDING USE OF AGENTS IN
ANIMAL MODELS AND THINGS IS
CAUSING OUR CENTER TO RE-EXAMINE
WHAT THE INNOVATIONS WOULD BE
AND I'M ADVOCATING FOR
INNOVATION IN CLINICAL TRIALS IN
WAYS THAT MAY OVERCOME OTHER
CHALLENGES THAT FACE NCI.
THANKS.
>> THANK YOU.
ANY OTHER COMMENTS OR DISCUSSION
POINTS?
ALL RIGHT.
WE NEED TO VOTE THEN.
EVERYONE IN FAVOR OF THIS
INITIATIVE PLEASE RAISE YOUR
HAND.
ANYONE OPPOSED?
ALL RIGHT.
MOTION PASSES.
THANK YOU VERY MUCH.
ALL RIGHT.
ANY OTHER ITEMS TO BE DISCUSSED
TODAY?
>> I HAD A QUESTION QUESTION FOR
SHEILA, WITH THE THREE
INITIATIVES PROCESS FOR
IDENTIFIED APPROPRIATE PEOPLE ON
CTAC AND EXPERTISE THAT MAY NOT
BE PART OF CTAC?
>> YES.
DEPENDING ON THE PARTICULAR
NEEDS OF THE GROUP THERE WILL BE
CTAC MEMBERS AND INDIVIDUALS
PLUS THE BROAD COMMUNITY WITH
THE APPROPRIATE EXPERTISE
IDENTIFIED TO PARTICIPATE.
IT WILL BE MRICHLD.
CLEARLY WE THESE TO BRING IN
ADDITIONAL EXPERTISE AND THIS IS
ONE OF THE THINGS THAT MAKES
THIS MECHANISM VALUABLE AND
ALLOWS US TO BRING IN EXPERTISE
TO ADVISE ON THINGS AND THEN
REPORT BACK TO CTAC ON WHAT WE
LEARN.
>> ALL RIGHT.
ANY OTHER DISCUSSION POINTS?
SHEILA, ANYTHING FROM YOU?
>> NO, OTHER THAN TO SAY THANK
YOU ALL VERY MUCH.
I KNOW IT'S BEEN A CHALLENGING
TIME.
MANY HAVE EXTENDED YOUR STAY ON
CTAC AND I GREATLY PATIENT THAT
AND WE'RE TRYING TO MOVE FORWARD
WITH POPULATING THE COMMITTEE.
WE HOPE TO HAVE A MEETING IN
PERSON AND RECOGNIZING MAYBE
ONCE A CAREER RATHER THAN ALL
VIRTUALLY IS VERY VALUABLE.
OUR NEXT MEETING IS NOVEMBER 19.
>> NOVEMBER 19 IS THE NEXT
MEETING AND I APPRECIATE
EVERYONE'S HELP WITH THE TOPICS
AND THEY'RE VERY IMPORTANT TO
OUR PATIENTS AS WELL AS OUR US.
THANK YOU FOR ALL YOUR TIME AND
EFFORT.
AND THEY DO GIVE YOU NEW MEETING
DATES IN THE INFORMATION SENT
OUT SO PUT THAT ON YOUR CALENDAR
FOR THE FUTURE AND THANKS
EVERYBODY.
WE'LL END THE MEETING THEN.
THANK YOU.