[AUDIO WILL START MOMENTARILY]
BEFORE WE GET STARTED, I WANT TO
MENTION THAT SAME DAY THAT THE
FINAL RULE FOR FDAAA WAS
PUBLISHED.
NIH PUBLISHED THE POLICY ON
CLINICAL TRIAL INFORMATION.
THERE ARE A LOT OF OVERLAPS
BETWEEN THESE TWO POLICIES.
THE NIH APPLIES TO ALL NIH
FUNDED AWARDEES AND
INVESTIGATORS CONDUCTING
CLINICAL TRIALS THAT ARE FUNDED
IN WHOLE OR PART BY NIH.
THAT POLICY COVERS ALL CLINICAL
TRIALS REGARDLESS OF STUDY,
PHASE, TYPE OF INTERVENTION OR
WHETHER SUBJECT TO FDAAA.
FOR MORE INFORMATION YOU CAN USE
THE URL LISTED HERE.
SO FOR TODAY'S AGENDA, WE'LL
FOCUS ON BACKGROUND AND FINAL
RULE PUBLICATION.
AND THEN GIVE SOME OVERVIEW OF
KEY PROVISIONS IN THE FINAL
RULE.
FIRST, WE'LL HAVE A BRIEF
SUMMARY OF KEY FINAL RULE
PROVISIONS, HOW TO DETERMINE
WHETHER OR NOT YOUR STUDY IS AN
APPLICABLE CLINICAL TRIAL.
EXPLANATION OF THE EFFECTIVE
DATE AND COMPLIANCE DATE,
PROVISIONS IN THE FINAL RULE.
APPLICABILITY, WHICH TRIALS HAVE
TO FOLLOW THE FINAL RULE
REQUIREMENTS FOR REGISTRATION
AND RESULTS SUBMISSION.
AND COMMENTS ABOUT UNAPPROVED
PRODUCTS AND THE RESULTS
[INDISCERNIBLE].
FIRST SOME BACKGROUND, JUST TO
SET THE STAGE FOR THESE
PROVISIONS.
AS MANY OF YOU PROBABLY HEALTH
RECORD KNOW, THERE ARE MANY
REASONS FOR REGISTRATION AND
RESULTS REPORTING REQUIREMENTS.
THESE INCLUDE HUMAN SUBJECTS,
PROTECTION CONCERNS IMNUMERATED
HERE.
ISSUES ABOUT RESEARCH INTEGRITY.
CONCERN ABOUT THE NEED TO
PRACTICE EVIDENCE BASED
MEDICINE, AND THE NEED TO HAVE A
RATIONALE METHOD OF ' RESEARCH
RESOURCES.
HOWEVER, THESE ALL OBSCURE THE
FACT THAT REPORTING THE RESULTS
IS WHY STUDIES ARE CONDUCTED IN
THE FIRST PLACE.
AND WITHOUT RESULTS REPORTING,
THERE IS GENERALLY NO POINT IN
DOING THE STUDY.
I THINK THIS POINT IS CENTRAL.
THE FININGAL RULE REPRESENTS A
PARADIGM SHIFT, TRADITIONALLY
INVESTIGATORS DECIDED WHETHER,
WHEN AND HOW TO REPORT THEIR
RESULTS.
AS A RESULT AS WE ALL KNOW, MANY
STUDIES ARE NEVER REPORTED, UP
TO A THIRD OF NIH FUNDED
STUDIES.
SIMILAR PROPORTIONS IN OTHER
DOMAINS.
AND FOR THOSE STUDIES THAT WERE
PUBLISHED, THERE WAS RAMPANT
CHERRY PICKING OF OUTCOME
MEASURES AND 59 VERSE
VOLUNTEERS.
UNDER FDAAA AND THE NIH POLICY
REGISTRATION AND RESULTS
REPORTING MUST BE DONE ON A
LEGALLY DEFINED TIMELINE.
NO LONGER LEFT TO THE WHIM OF
THE INVESTIGATOR.
IN ADDITION, ORGANIZATIONS THAT
SPONSOR STUDIES ARE BE HELD
RESPONSIBLE.
SO THIS IS GOING TO REQUIRE
FUNDAMENTAL CHANGES THROUGHOUT
THE CLINICAL RESEARCH
ENTERPRISE.
FUNDERS, SPONSORS, AND
INVESTIGATORS WILL ALL HAVE TO
CHANGE HOW THEY CONDUCT THEIR
ACTIVITIES.
AS A RESULT, THERE WILL BE
GREATER TRANSPARENCY INTO HUMAN
EXPERIMENTATION.
AND THIS IS IMPORTANT.
TIME TO DECIDE IF A STUDY IS
WORTH REPORTING IS BEFORE THE
PARTICIPANTS ARE PUT AT RISK,
NOT AFTER.
THIS IS JUST A BRIEF OVERVIEW OF
3 REPORTING REQUIREMENTS THAT
ARE RELEVANT IN THE UNITED
STATES.
ICMJE STANDS FOR THE
INTERNATIONAL COMMITTEE OF
JOURNAL EDITORS, THE FDAAA FINAL
RULE IS THE MAIN TOPIC OF THIS
WEBINAR AND THE FINAL NIH
POLICY.
AS YOU CAN SEE, THE POLICY
COVERS TRIAL REGISTRATION ONLY.
AND THE OTHER 2 POLICIES COVER
REGISTRATION AND RESULTS
REPORTING.
THE ICMJE POLICY COVERS ALL
CLINICAL TRIALS REGARDLESS OF
PHASE, INTERVENTION TYPE,
FUNDING SOURCE.
AND THE ENFORCEMENT PROVISIONS
ARE BASICALLY THAT EACH EDITOR
CAN REFUSE TO PUBLISH THE
MANUSCRIPT.
FDAAA COVERS -- COVERS CLINICAL
TRIALS THAT INVOLVE DRUGS,
BIOLOGICALS OR DEVICES RATED BY
THE FDA THAT ARE NOT PHASE I OR
THE EQUIVALENT FOR DEVICES.
CORES THESE TRIALS REGARDLESS OF
FUNDING SOURCE.
THE ENFORCEMENT IS BROAD,
CRIMINAL PROCEEDINGS, CIVIL
MONRY PENALTIES OR LOSS OF
FUNDING INCLUDING NIH FUNDING.
THE NIH POLICY COFFERS RESULTS
REPORTING, AND COVERS TRIALS OF
ANY PHASE, ANY INTERVENTION TYPE
THAT ARE FUNDED IN WHOLE OR PART
BY NIH.
THE ENFORCEMENT IS BASICALLY THE
POTENTIAL FOR LOSS OF NIH
FUNDING.
OF NOTE UNDER BOTH FDAAA AND
NIH, THE LOSS OF FUNDING COULD
THEORETICALLY APPLY TO THE
INSTITUTION, THE SPONSOR
INSTITUTION OR GRANTEE
INSTITUTION, NOT JUST THE
INDIVIDUAL RESEARCHER.
MANY PEOPLE WONDER WHY RESULTS
REPORTING IS IMPORTANT, EVEN
WHEN THEY HAVE A PUBLICATION.
AND I'M JUST USING THIS
PUBLICATION FROM THE "NEW
ENGLAND JOURNAL OF MEDICINE" AS
AN EXAMPLE.
IN THIS PARTICULAR PUBLICATION,
THEY NOTE THE FOLLOWING.
THE PROTOCOL, 27 SECONDARY
OUTCOME MEASURES.
THEY REPORT DATA FOR 19 OF THESE
HERE, AND IN THE SUPPLEMENTARY
APPENDIX BUT LINK TO CLINICAL
TRIALS.GOV FOR THE 8 OUT COME
MEASURES WHICH ARE NOT REPORTED
HEAR.
YOU COULD SEE HOW THIS WOULD
WORK.
THIS SHOWS A USEFUL WAY TO USE
THE CLINICAL TRIALS.GOV RESULTS
DATABASE TO COMPLEMENT A
PUBLICATION.
SO NOW FOR THE TIMELINE OF
FDAAA.
IT WAS ENACTED EXACTLY NINE
YEARS AGO, SEPTEMBER, 2007.
THE BASIC PROVISIONS TOOK EFFECT
OVER THE NEXT COUPLE YEARS.
BUT NOTICE A PROPOSE RULE MAKING
FOR THESE REGULATIONS WAS ISSUED
IN NOVEMBER OF 2014, AND THAT
CAME WITH A PUBLIC COMMENT
PERIOD THAT ENDED IN MARCH,
2015.
AND THE FINAL RULE WAS PUBLISHED
LAST WEEK.
OF NOTE, WE RECEIVED
APPROXIMATELY 900 COMMENTS FROM
A VERY DIVERSE GROUP OF PEOPLE
INCLUDING THOSE ENTITIES LISTED
HERE.
THE FINAL RULE PREAMBLED
DISCUSSED HOW THESE COMMENTS
INFORMED THE RULE.
HERE IS THE FINAL RULE.
DETAILS OF THIS CAN BE FOUND IN
THIS RECENT ARTICLE FROM LAST
WEEK IN THE NEW ENGLAND JOURNAL
OF MEDICINE, WHICH WE'LL GO INTO
MORE DETAIL AND MORE EXPLANATION
THAN WE WILL BE DOING HERE.
SO I'LL BRIEFLY GIVE YOU
OVERVIEW OF KEY PROVISIONS.
BUT FIRST, LET ME NOTE THAT MUCH
OF THIS WEBINAR WILL FOCUS ON
CHANGES BETWEEN FDAAA OR BETWEEN
THE STATUTE AND THE FINAL RULE.
FOR THOSE WHO WANT MORE
[INDISCERNIBLE] YOU CAN SEE THIS
PART OF OUR CLINICAL TRIALS.GOV
WEBSITE, THE LINK IS ON THE
LEFT.
THE SCREEN SHOT IS ON THE RIGHT.
ALSO, WE NOTE THAT PRS INFO
SITE, THE URL IS LISTED WHERE
WE'LL CONTINUALLY POST FINAL
RULE INFORMATION AND OTHER
RESOURCES.
SO SOME KEY PROVISIONS, TO GIVE
AN OVERVIEW, BEFORE DR. WILLIAMS
GOES INTO MORE DETAIL.
FIRST, REQUIRES REGISTRATION AND
RESULTS SUBMISSION FOR
APPLICABLE CLINICAL TRIALS OR
WHAT WE CALL ACT SAID.
IN ADDITION, IT ALLOWS FOR
AUTHORIZATION OF POSTING OF
REGISTRATION INFORMATION FOR
DEVICE TRIALS OF UNAPPROVED OR
UNCLEAR PRODUCTS.
WE'VE REFERRED TO THIS AS THE
DEVICE LOCK BOX, AND WHAT THIS
SAYS, FINAL ROOM IT WILL BE
POSSIBLE FOR RESPONSIBLE PARTIES
TO ESSENTIALLY OPT OUT OF THE
LOCK BOX TO ENABLE THE PUBLIC
POSTING OF THEIR REGISTRATION,
IF THEY SO DESIRE.
THE FINAL RULE CLARIFIES AND
EXPANDS THE REGISTRATION DATA
ELEMENTS, EXPANDS THE SCOPE OF
RESULTS REPORTING TO INCLUDE
TRIALS OF UNAPPROVED PRODUCT.
AS MANY OF YOU KNOW, THE BASIC
RESULTS REQUIREMENTS IN EFFECT
PRIOR TO THE FINAL RULE REQUIRED
THE REPORTING OF RESULTS FOR
ACTS OF TRIALS OF APPROVED DRUG
AND DEVICE PRODUCTS, NOT FOR
TRIALS OFF UNAPPROVED DRUG AND
DEVICE PRODUCTS.
THIS FINAL RULE EXPANDS THOSE
REQUIREMENTS.
ALITIES CLARIFIES AND EXPANDS
RESULTS, CERTAIN RESULTS DATA
ELEMENTS.
OF NOTE, IT REQUIRES SUBMISSION
OF PROTOCOL AND STATISTICAL
ANALYSIS PLAN, A SEPARATE
DOCUMENT, AT THE TIME OF RESULTS
SUBMISSION.
THIS WOULD BE SUBMITTED IN THE
FORM OF A PDF WITH THE DETAILS
OF THE SUBMISSION REQUIREMENTS,
FOUND IN THE FINAL RULE.
THE FINAL RULE NOTES THAT THE
SUBMISSION OF NARRATIVE
SUMMARIES ARE NOT REQUIRED.
AND IT ALSO REVISES THE QUALITY
CONTROL AND POSTING PROCESS.
SO LET'S JUST TALK FOR A FEW
MINUTES ABOUT THE DETERMINATION
OF AN ACT OR APPLICABLE CLINICAL
TRIAL.
THESE PROVISIONS APPLY TO TRIALS
THAT ARE INITIATED AFTER THE
EFFECTIVE DATE, JAN 18, 2017.
WHAT'S INVOLVED IS THAT PREVIOUS
DATE OR AMBIGUOUS LANGUAGE IN
THE STATUTE HAVE BEEN REPLACED,
IF YOU WILL, BY MORE CONCRETE
OBJECTIVE CRITERIA, EACH OF
WHICH HAS AN ASSOCIATED DATA
ELEMENT, SOME OF WHICH ARE NEW.
SO THE DATA ELEMENTS WHICH
TOGETHER CAN FORM A CHECKLIST
INCLUDE WHETHER OR NOT THE STUDY
TYPE IS INTERVENTIONAL, WHETHER
THE STUDY INCLUDES ONE OR MORE
U.S. FDA REGULATED DRUG OR U.S.
FDA REGULATED DEVISE PRODUCTS.
IT INCLUDES THE PROVISION THAT
THE STUDY PHASE IS NOT PHASE I
FOR DRUGS, OR THAT THE PRIMARY
PURPOSE IS NOT DEVISE
FEASIBILITY.
THAT'S BY THE WAY A NEW MENU
ITEM FOR PRIMARY PURPOSE THERE.
IT ALSO REQUIRES THAT ONE OF THE
FOGGY PLY FOR THE TRIALS TO BE
CONSIDERED.
THESE ALL HAVE ASSOCIATED DATA
ELEMENTS AS WELL.
ONE OR MORTALITY HAS TO BE
LOCATED IN THE U.S.
THERE HAS TO BE A U.S. FDA IND
OR IDE NUMBER.
OR THE PRODUCT NEEDS TO BE
MANUFACTURED IN THE U.S. AND
EXPORTED FOR THE USE OF
RESEARCH.
THESE ARE SPECIFIC DATA ELEMENTS
EXPLAINED IN MORE DETAIL IN THE
FINAL RULE.
BY THE WAY, IN THIS SLIDE AND
ALL THE OTHERS THAT DISCUSS
DETAILS ABOUT DATA ELEMENTS, THE
SPECIFIC PROVISION IN THE FINAL
RULE ARE LISTED AT THE BOTTOM
WHERE YOU'LL FIND MORE LENGTHY
EXPLANATION.
[TECHNICAL DIFFICULTIES]
>> MY GOAL IS TO TAKE A BIT OF A
STEP BACKWARDS, AND GIVE AN
OVERVIEW OF HOW THE FINAL RULE
ITSELF IS STRUCTURED.
AND HOW PEOPLE CAN NAVIGATE
INFORMATION WITHIN THE FINAL
RULE ITSELF.
AS WELL AS [TECHNICAL
DIFFICULTIES] RELATED TO THE
EFFECTIVE DATE, THE COMPLIANCE
DATE, AND WHICH TRIALS ARE
APPLICABLE [TECHNICAL
DIFFICULTIES] SO HERE IS A BRIEF
SEVERE OVERNIGHT VIEW OF THE
FINAL RULE ITSELF.
IT'S IN 8 DIFFERENT SECTIONS
WITH ONE FINAL SECTION, WHICH IS
THE REGULATORY TEXT WHICH IS THE
ACTUAL REGULATIONS THAT YOU CAN
FIND AT 42 CFR 11.
IN TERMS OF THE STRUCTURE AND
[INAUDIBLE] WE REFER TO
GENERALLY AS THE PREAMBLE.
A GENERAL IDEA FOR THE PREAMBLE
IS TO GET INSIGHT INTO THE
REQUIREMENTS ELUCIDATED IN THE
[INAUDIBLE].
YOU CAN SEE THAT WE PROVIDED A
LITTLE BIT OF BACKGROUND, AS
WELL AS AN OVERVIEW OF THE
STATUTORY PROVISIONS, THE
PROVISIONS THAT [INAUDIBLE], AND
THEN WE INTO MORE DETAIL ABOUT
THE PUBLIC COMMENTS WE RECEIVED
AS WELL AS HIGHLIGHTING SOME OF
THE KEY ISSUES.
ONE OF THOSE KEY ISSUES THAT
WE'LL BE COVERING IS THE
SUBMISSION OF RESULTS
INFORMATION FOR TRIALS OF
[INDISCERNIBLE].
SECTIONS A THROUGH E, WITHIN
THAT SECTION 4, ARE GOING IN TO
MORE DETAIL OF EACH OF THE
SECTIONS OF THE REGULATIONS
THEMSELVES.
AND THERE IS A -- WITHIN THE
LAST SECTION WITHIN THERE IS F,
WHICH IS THE EFFECTIVE DATE,
COMPLIANCE DATE AND
APPLICABILITY OF REQUIREMENTS IN
THIS PART.
AND THAT'S REALLY WHAT I'LL BE
FOCUSING ON TODAY.
THAT -- THERE IS A LOT OF
ELABORATION IN THE PREAMBLE.
WE'LL TRY TO BREAK IT DOWN FOR
YOU SO YOU CAN UNDERSTAND THAT
ASPECT OF IT.
WITHIN THAT SECTION ALSO, JUST
AS A [INAUDIBLE] DISCUSSED THE
PUBLIC COMMENTS.
[POOR AUDIO].
WE SUMMARIZED THE PUBLIC
COMMENTS AND PROVIDE RESPONSES
TO THOSE COMMENTS WHICH SHOULD
GIVE YOU INSIGHT INTO OUR
DECISION MAKING THAT GOES TO
THAT THIRD SECTION, WHICH IS TO
TRY [INDISCERNIBLE] WHAT THE
FINAL RULE IS REQUIRING.
SO THEN WHEN YOU LOOK AT THE
REGULATORY TEXT ITSELF, IT'S
BROKEN DOWN INTO SUBPARTS A
THROUGH E.
WE'RE NOT GOING TO WALK THROUGH
EACH OF THESE IN ORDER TODAY BUT
AS YOU SAW WITH THE OUTLINE FOR
THE WEBINARS, THE OVERALL -- THE
NEXT WEBINAR WILL REALLY FOCUS
ON THE REGISTRATION ASPECTS, SO
SUBPART A AND B.
SO FOCUSING ON WHO HAS TO SUBMIT
INFORMATION, WHICH TRIALS MUST
BE REGISTERED, DEBORAH TOUCHED
BRIEFLY ON THE APPROACH FOR
DETERMINING AN APPLICABLE
CLINICAL TRIAL UNDER THE FINAL
RULE.
WE'LL COVER THAT AGAIN IN THE
SECOND WEBINAR BUT WE'LL ALSO
FOCUS IN MORE DETAIL ON WHICH
CLINICAL TRIAL REGISTRATION
INFORMATION MUST BE SUBMITTED
AND HOW SOME OF THE REQUIREMENTS
ARE A LITTLE BIT DIFFERENT FROM
[INDISCERNIBLE] [POOR AUDIO]
TODAY I WILL BE TOUCHING ON TWO
ASPECTS OF THESE, IN TERMS OF
WHICH TRIALS SHOULD HAVE RESULTS
INFORMATION SUBMITTED BASED ON
THE NEW FINAL RULE REQUIREMENTS,
KIND OF LAYERING THE EFFECT
[POOR AUDIO] DESCRIBES THE
POTENTIAL LEGAL CONSEQUENCES OF
NON COMPLIANCE.
SO I'M GOING TO GO AHEAD AND
JUMP IN AND TALK ABOUT THE
EFFECTIVE DATE, COMPLIANCE DATE,
AND APPLICABILITY ISSUES.
AND I JUST WANT TO CHECK ONE
CLEARLY.
ONE OF THE KEY ISSUES ONE OF THE
KEY ISSUES, WHEN DO THESE THINGS
START KICKING IN AND WHEN DO I
ACTUALLY HAVE TO START PAYING
ATTENTION AND CHANGING MY
PRACTICES IN TERMS OF
REGISTRATION AND RESULTS
REPORTING?
SO ONE OF THE THINGS THAT THE
FINAL RULE DID WAS LAY OUT WHAT
TERM THE EFFECTIVE DATE AND
COMPLIANCE DATE.
THE EFFECTIVE DATE WAS DESCRIBED
IN THE PREAMBLE SECTION OF THE
FINAL RULE AS BEING JANUARY 18,
2017.
WHICH IS ABOUT 120 DAYS FROM THE
PUBLICATION OF THE FINAL RULE IN
THE FEDERAL REGISTER.
AS MANY OF YOU WILL RECALL, THE
NOTICE OF PROPOSED RULE MAKING
HAD PROPOSED A 45 DAY EFFECTIVE
DATE.
WE RECEIVED MANY PUBLIC COMMENTS
ON THIS PARTICULAR PROPOSAL.
AND MANY OF THE RESPONDENT'S WHO
WERE OPPOSED TO THE 45 DAY
EFFECTIVE DATE RAIDED ISSUES
ABOUT THE TIME THAT IT WOULD
TAKE TO UNDERSTAND THE FINAL
RULE REQUIREMENTS, AND ADAPT
TO -- ADAPT, TRAIN, AND UPDATE
SYSTEMS AND PROCESSES AND
PROCEDURES IN ORDER TO BE ABLE
TO MEET THE NEW REQUIREMENTS OF
THE FINAL RULE.
AND WE HEARD THOSE COMMENTS.
ANOTHER GOOD ASPECT IS THAT IT
ALSO HELPED TO ENSURE THAT THE
SYSTEM AT CLINICAL TRIALS.GOV
WILL BE FULLY READY TO
ACCOMMODATE THESE REQUIREMENTS
AS OF THE EFFECTIVE DATE.
I'LL TALK IN A BIT ABOUT SOME OF
OUR GENERAL PLANS FOR HOW WE
WILL BE IMPLEMENTING THIS WITHIN
THE PROTOCOL REGISTRATION AND
RESULTS SYSTEM, ITSELF.
THE SECOND ASPECT IS THE
COMPLIANCE DATE, DEFINED AT 90
DAYS AFTER THE EFFECTIVE DATE,
OR APRIL 18, 2017.
SO BASICALLY, THAT DATE IS THE
MAXIMUM PERIOD OF TIME THAT
RESPONSIBILITY PARTIES HAVE TO
COME INTO COMPLIANCE WITH THE
FINAL RULE REQUIREMENT.
SO THE REQUIREMENTS WILL BE
EFFECTIVE AS OF JANUARY 18, BUT
THE EXPECTATION IS THAT
RESPONSIBLE PARTIES WILL BE
MEETING ALL OF THE FINAL RULE
REQUIREMENTS AS OF APRIL 18,
2017.
WITHIN THE APPLICABILITY
SECURITIES OF THE FINAL --
SECTION OF THE FINAL RULE, WE'VE
PROVIDED A SPECIFIC PAGE NUMBER.
THERE IS A TABLE THAT TRIES TO
WALK THROUGH THE DIFFERENT
ASPECTS OF THE APPLICABILITY OF
THE REQUIREMENTS.
I AM NOT GOING TO TALK FROM THIS
TABLE SPECIFICALLY, BUT WANT TO
ENSURE THAT YOU HAVE IT AT YOUR
FINGER TIPS FOR REFERENCE, AND
BASICALLY, IN THE SUBSEQUENT
SLIDES I'M GOING TO BE WALKING
THROUGH THE PRACTICAL
IMPLICATIONS OF THE WAY THAT
THIS IS ALL DEFINED.
BUT GENERALLY SPEAKING, THE
APPROACH THAT'S TAKEN IN THE
FINAL RULE IS A LITTLE BIT
DIFFERENT THAN WHAT WAS PROPOSED
IN THE NOTICE OF PROPOSE RULE
MAKING IN TERMS OF THE APPLICANT
OF THE FINAL RULE REQUIREMENTS.
THE GENERAL IDEA WAS TO COME UP
WITH A SIMPLER, MORE STREAMLINED
APPROACH THAT WOULD BE AS EASY
TO UNDERSTAND AS POSSIBLE, BOTH
FOR US ADMINISTRATORS HERE AT
CLINICAL TRIALS.GOV AS WELL AS
FOR RESPONSIBLE PARTIES WHO ARE
INTENDING TO FOLLOW THE FINAL
RULE REQUIREMENTS.
THERE ARE TWO THINGS TO KEEP IN
MIND, IN TERMS OF WHICH
REQUIREMENTS APPLY.
AND WHEN WE TALK ABOUT WHICH
REQUIREMENTS, WE'RE EITHER
REFERRING TO THE NEW
REQUIREMENTS WITHIN THE FINAL
RULE, OR WE'RE REFERRING TO THE
EXISTING REQUIREMENTS THAT ARE
WITHIN THE STATUTE OR THE
EXISTING LAW OF THE FOOD AND
DRUG ADMINISTRATION AMENDMENTS
ACT.
FOR REGISTRATION INFORMATION IN
TERMS OF DETERMINING WHICH
INFORMATION MUST BE SUBMITTED TO
CLINICAL TRIALS.GOV, THAT'S
GOING TO BE DETERMINED BASED ON
THE STUDY START DATE, OR WHEN
THE TRIAL WAS INITIATED.
AND SO IF THE TRIAL IS INITIATED
ON OR AFTER JANUARY 18 OF 2017,
THAT'S THE EFFECTIVE DATE OF THE
FINAL RULE, A RESPONSIBLE PARTY
MUST FOLLOW THE FINAL RULE
REQUIREMENTS.
IF THE STUDY START DATE IS
BEFORE JANUARY 18, THEN THE
RESPONSIBLE PARTY WOULD CONTINUE
TO FOLLOW THE EXISTING
REQUIREMENTS THAT ARE IN PLACE
UNDER THE STATUTE.
AND UNDERNEATH THAT BULLET YOU
CAN SEE THAT THERE IS OTHER
QUALIFICATIONS BASED ON WHEN THE
LAW, ITSELF, WAS PASSED BACK IN
SEPTEMBER, 2007.
THAT ALSO DETERMINES WHICH
TRIALS COME UNDER THE SCOPE OF
THE LAW.
FOR RESULTS TOGETHER, THE -- FOR
DETERMINING WHICH REQUIREMENTS
MUST BE FOLLOWED, IT'S GOING TO
BE DETERMINED BY THE PRIMARY
COMPLETION DATE OF THAT CLINICAL
TRIAL.
SO IF THE PRIMARY COMPLETION
DATE IS ON OR AFTER THE
EFFECTIVE DATE OR JANUARY 18,
2017, THEN THE FINAL RULE
REQUIREMENTS WILL BE FOLLOWED
FOR RESULTS INFORMATION.
BUT IF THAT PRIMARY COMPLETION
DATE IS BEFORE JANUARY 18, THE
BASIC REQUIREMENTS THAT ARE
WITHIN THE STATUTE WOULD
CONTINUE TO BE FOLLOWED BY
RESPONSIBLE PARTIES.
THERE IS A LOT OF DIFFERENT
IMPLICATIONS FOR THIS.
AND WE'LL GO INTO MORE DETAILS
ABOUT EACH OF THESE.
BUT ONE OF THE THINGS THAT I
WANT TO REFER BACK TO ARE SOME
OF THE DEFINITIONS THAT ARE USED
FOR DETERMINING THIS IMPORTANT
DATE.
WE HAVE THE STUDY START DATE
DEFINITION, THEN WE ALSO HAVE
THE PRIMARY COMPLETION DATE
DEFINITION, UNDERSTANDING THESE
DEFINITIONS ARE REALLY KEY FOR
ENSURING THAT IT'S CLEAR WHICH
THINGS HAVE TO BE FOLLOWED FOR
THE APPLICABLE CLINICAL TRIAL.
SO BASED ON THAT OUTLINE OF THE
TABLE OF CONTENTS, THE
DEFINITIONS FOR MUCH OF THE RULE
ARE CONTAINED 42CF11.10.
TO GO ALONG WITH THAT STUDY
START DATE DEFINITION, THERE IS
A FURTHER ELABORATION OF THE
TERM ENROLLED, THAT CONCEPT
ENROLL OR ENROLLED HAS ITS OWN
DEFINITION IN 42 CFR 11.10.
THE WAY THAT THAT'S DEFINED, YOU
CAN READ IT ON THE SLIDE HERE.
BUT BASICALLY, IT'S A HUMAN
SUBJECTS AGREEMENT TO
PARTICIPATE IN THE CLINICAL
TRIAL FOLLOWING COMPLETION OF
THE INFORMED CONSENT PROCESS AS
IT RELATES TO OTHER REGULATIONS
THAT APPLY.
SOME -- AND WE ALSO FURTHER
ELABORATED THAT POTENTIAL
SUBJECT WHOSE ARE SCREENED FOR
THE PURPOSE OF DETERMINING
ELIGIBILITY, BUT DO NOT ACTUALLY
PARTICIPATE IN THE CLINICAL
TRIAL, ARE NOT CONSIDERED TO BE
ENROLLED OR TO MEET THAT
DEFINITION OF ENROLLED UNLESS
FOR SOME REASON THE PROTOCOL,
ITSELF, HAD SPECIFIED OTHERWISE.
SO THE OTHER KEY DEFINITION FOR
UNDERSTANDING THE APPLICABILITY
REQUIREMENTS AS THE PRIMARY
COMPLETION DATE, AGAIN, THIS
DEFINITION IS AVAILABLE IN 42
CFR 11.10.
IT IS BASED ON THE DEFINITION OF
COMPLETION DATE IN THE STATUTE.
AND WE FURTHER ELABORATED ON A
COUPLE KEY ASPECTS OF THE
DEFINITION.
SO GENERALLY SPEAKING, THE
PRIMARY COMPLETION DATE IS THE
DATE THAT THE FINAL SUBJECT WAS
EXAMINED OR RECEIVED AN
INTERVENTION FOR THE PURPOSE OF
FINAL DATA COLLECTION FOR THE
PRIMARY OUTCOME.
ONE OF THE REASONS THAT WE
MODIFIED THE NAME FROM
COMPLETION DATE TO PRIMARY
COMPLETION DATE WAS TO REALLY
CALL ATTENTION TO THE FACT THAT
THIS IS RELATED TO FINAL DATA
COLLECTION FOR THE PRIMARY
OUTCOME MEASURE AS COMPARED TO
THE TRADITIONAL DEFINITION OF
STUDY COMPLETION, WHICH MAY
INCLUDE KIND OF MORE BROADLY
COLLECTION OF ALL INFORMATION OF
PRIMARY OUTCOME MEASURES,
SECONDARY, AND ADVERSE EVENT
INFORMATION.
THERE ARE CERTAIN SITUATIONS IN
WHICH A CLINICAL TRIAL MAY HAVE
MORE THAN ONE PRIMARY OUTCOME
MEASURE.
THOSE PRIMARY OUTCOME MAY HAVE
FINAL DATA COLLECTION ON
DIFFERENT DATES. SO THE
QUESTION HAD ALWAYS BEEN, WHICH
DATE DO WE USE FOR TERMINATION
OF THE PRIMARY COMPLETION DATE?
AND THE FINAL RULE CLARIFIES
THAT IF THERE ARE MULTIPLE
PRIMARY OUTCOME MEASURES, THAT
IT'S THE DATE ON WHICH DATA
COLLECTION WAS COMPLETED FOR ALL
OF THE PRIMARY OUTCOMES.
AND JUST AS WITH THE STUDY START
DATE INFORMATION IS FIRST
ENTERED AS ESTIMATED FOR PRIMARY
COMPLETION DATE, AND THEN
UPDATED TO ACTUAL -- WHEN THE
ACTUAL COMPLETION DATE OCCURS.
IN FUTURE WEBINARS WHEN WE TALK
IN MORE DETAIL ABOUT BOTH
REGISTRATION REQUIREMENTS AND
RESULTS REQUIREMENTS, WE'LL GO
INTO A LITTLE BIT MORE DETAIL
ABOUT THE UPDATE OBLIGATIONS AND
WHEN THOSE ACTIVITIES HAVE TO
OCCUR.
SO SOME OF THE PRACTICAL
IMPLICATIONS OF TRYING TO FIGURE
OUT WHAT APPLIES FOR YOUR
CLINICAL TRIAL.
JUST REINFORCING, AGAIN, THAT
THE KEY THINGS TO REALLY FOCUS
ON ARE THE STUDY START DATE FOR
REGISTRATION INFORMATION, AND
THE PRIMARY COMPLETION DATE FOR
RESULTS INFORMATION.
THE DETERMINATION OF WHICH
REQUIREMENTS APPLY IS THEREFORE
INDEPENDENT OF WHEN THAT
INFORMATION WAS ACTUALLY
SUBMITTED TO CLINICAL
TRIALS.GOV.
IT'S STILL JUST BASED ON THOSE
DATA ELEMENTS OF STUDY START
DATE AND PRIMARY COMMOTION DATE.
I INCLUDED A COUPLE EXAMPLES ON
THIS SLIDE TO PLAY THROUGH SOME
OF THE SCENARIOS.
ONE EXAMPLE WOULD BE IF YOU HAD
A STUDY START DATE IN MARCH,
2017, WHICH WOULD BE AFTER THE
EFFECTIVE DATE OF THE RULE,
BUT -- IN ANTICIPATION OF
RECRUITING AND ALL THE OTHER
ACTIVITIES ASSOCIATED WITH THE
TRIAL, THAT TRIAL WAS ACTUALLY
FIRST REGISTERED ON CLINICAL
TRIALS.GOV IN DECEMBER OF 2016,
WHAT WOULD BASICALLY HAPPEN IS
THAT IN DECEMBER OF 2016 WHEN
THAT TRIAL IS FIRST REGISTERED,
BEFORE THE EFFECTIVE DATE, THE
RESPONSIBILITY PARTY WOULD
FOLLOW THE REQUIREMENTS THAT ARE
IN PLACE AT THE TIME OF
REGISTRATION, WHICH ARE
BASICALLY THE STATUTORY
REQUIREMENTS.
THEN BECAUSE THE STUDY START
DATE IS IN MARCH OF 2017, IT IS
ACTUALLY SUBJECT TO THE NEW
FINAL RULE REQUIREMENTS, SO OVER
THAT TIME PERIOD OF JANUARY TO
APRIL, 2016, IT WOULD BE
EXPECTED THAT THE RESPONSIBLE
PARTY WOULD UPDATE THE STUDY
RECORD TO MEET THE REQUIREMENTS
OF THE FINAL RULE.
ANOTHER EXAMPLE MIGHT BE A
CLINICAL TRIAL THAT HAD A START
DATE BACK IN JUNE OF 2014, THEN
COMPLETING IN JULY OF 2017.
SO THE PRIMARY COMPLETION DATE,
THEN, IS AFTER THE EFFECTIVE
DATE OF THE RULE.
BUT THE STUDY START DAY WAS
BEFORE THE EFFECTIVE DATE OF THE
RULE.
THERE IS ACTUALLY 2 DIFFERENT
PATHWAYS THAT ARE BEING
FOLLOWED, THE REGISTRATION
INFORMATION WILL CONTINUE TO
FOLLOW THE STATUTORY
REQUIREMENTS, BECAUSE THAT STUDY
START DATE WAS BEFORE JANUARY,
2017.
BUT THE RESULTS INFORMATION
WOULD FOLLOW THE FINAL RULE
REQUIREMENTS, BECAUSE THAT
PRIMARY COMPLETION DATE WAS
JULY, 2017, OR AFTER THE
EFFECTIVE DATE OF JANUARY, 2017.
THERE IS ALL SORTS OF SCENARIOS
WE COULD POSSIBLY RUN THROUGH
AND MAYBE WE CAN DO SOME OF THAT
AT THE END.
BUT -- SO THEN THE BIG QUESTION
IS HOW IS THIS GOING TO PLAY OUT
WITHIN THE PROTOCOL REGISTRATION
AND RESULTS SYSTEM AT CLINICAL
TRIALS.GOV IN TERMS OF HOW THIS
IS IMPLEMENTED, HOW THOSE DATA
ELEMENTS WILL BE AVAILABLE TO A
RESPONSIBLE PARTY.
RIGHT NOW, OUR PLANS ARE TO HAVE
A RELEASE TO WHAT WE CALL THE
TEST PRS, THE PRS TEST SYSTEM.
SO WHERE MID TO LATE NOVEMBER.
THAT WOULD HAVE ALL OF THE
REGISTRATION AND RESULTS FINAL
RULE DATA ELEMENTS.
AND THAT WOULD INCLUDE THE DATA
ELEMENT DEFINITION DOCUMENTS, AS
WELL AS XLM SCHEMA ASSOCIATED
WITH SUBMITTING THOSE DATA
ELEMENTS THEMSELVES.
THAT RELEASE 2PRS TEST WOULD SIT
THERE BASICALLY UNTIL THE
EFFECTIVE DATE OF THE FINAL
RULE, AT WHICH POINT IN TIME ON
JANUARY 18, WE WOULD ROLL THAT
RELEASE OUT INTO THE OPERATIONAL
PRS SYSTEM.
WE WOULD MAKE THOSE DATA
ELEMENTS THAT ARE NEWLY REQUIRED
BY THE FINAL RULE AVAILABLE.
AND WE WOULD ALSO START
IMPLEMENTING WARNINGS IF THOSE
NEW ELEMENTS WERE NOT COMPLETED,
BUT WE WOULDN'T NECESSARILY HAVE
THOSE DATA ELEMENTS BE ERRORS TO
ALLOW FOR THE TIME PERIOD
BETWEEN THE EFFECTIVE DATE AND
THE COMPLIANCE DATE.
AND THEN ALSO, TAKING INTO
FURTHER ACCOUNT THE FACT THAT
THE NEW REGISTRATION
REQUIREMENTS, AS WELL AS THE NEW
RESULTS REQUIREMENTS, ARE BASED
ON STUDY START DATE AND BASED ON
THE PRIMARY COMPLETION DATE.
THOSE WARNINGS AND EVENTUALLY
ERRORS WOULD BE TIED TO THOSE
DATES AS WELL.
SO WE WOULD BE TAKING INTO
ACCOUNT BOTH THE STUDY START
DATE AND PRIMARY COMPLETION
DATE, IN TERMS OF DETERMINING
WHICH DATA ELEMENTS ARE GOING TO
BE REQUIRED FOR A PARTICULAR
CLINICAL TRIAL.
AND SO THEN ON APRIL 18, THE
GENERAL PLAN RIGHT NOW IS FOR
THOSE DATA ELEMENTS THAT WOULD
BE NEWLY REQUIRED BY THE FINAL
RULE, TO START HAVING THOSE SHOW
UP AS ERRORS, IF THEY ARE NOT
COMPLETED.
THIS GENERAL APPROACH IS
INTENDED, ONE, BY HAVING THE
INFORMATION AVAILABLE ON PRS
TEST FOR A PERIOD OF TIME TO
GIVE PEOPLE AN ADEQUATE
OPPORTUNITY TO SEE THE NEW DATA
ELEMENTS, UNDERSTAND HOW THEY'RE
GOING TO BE IMPLEMENTED WITHIN
CLINICAL TRIALS.GOV, AND FOR
ALSO US TO WORK OUT ANY BUGS
THAT MIGHT OCCUR WITH THAT
PARTICULAR IMPLEMENTATION.
THE IDEA, THEN, IS BY HAVING
THAT EXTENDED PERIOD OF TIME ON
PRS TESTS, THEN EVERYONE WILL
HAVE HAD ADEQUATE FAMILIARITY.
THEN ALSO HAVING A PHASED IN
APPROACH ON THE PRS WILL ALLOW
PEOPLE SUFFICIENT TIME TO GET
CAUGHT UP TO SPEED WITH ALL OF
THE NEW REQUIREMENTS FOR THOSE
STUDIES THAT ARE SUBJECT TO
THEM.
I AM ALSO GOING TO BRIEFLY
MENTION, THOUGH, THAT PRIOR TO
THAT PRS TEST RELEASE IN THE --
IN NOVEMBER, THAT WE HAVE AN
UPCOMING RELEASE ON PROTESTS
THAT WILL BE HAPPENING THIS
WEEK.
THAT RELEASE IS PRIMARILY
INCLUDING ADDITIONAL OPTIONAL
DATA ELEMENTS RELATED TO THE
SUBMISSION OF CLINICAL TRIAL
RESULTS INFORMATION.
WE'LL TALK ABOUT SOME OF THESE
DATA ELEMENTS IN THE CONTEXT OF
THE FINAL RULE, BUT RIGHT NOW,
MANY OF THESE ARE OPTIONAL IN
THE WAY THAT THEY'LL BE MADE
AVAILABLE WITHIN THE SYSTEM.
THERE ARE DIFFERENT STUDY
DESIGNS THAT WILL INCORPORATE A
UNIT OF ASSIGNMENT OR ANALYSIS,
THAT IS SOMETHING OTHER THAN
PARTICIPANTS, MEANING THAT A
UNIT OF EYES OR LESIONS OR KNEES
AS BEING EVALUATED.
AND RIGHT NOW WE DON'T HAVE
NECESSARILY A STRUCTURED WAY TO
ACCOMMODATE THAT INFORMATION,
BUT THERE IS LESS STRUCTURED
WAYS IN WHICH PEOPLE CAN SUBMIT
THAT INFORMATION.
SO WHAT WE'VE ROLLED INTO THIS
NEXT RELEASE IS ADDITIONAL
OPTIONS FOR PEOPLE TO BE ABLE TO
DESCRIBE WHEN THE UNITS OF
ASSIGNMENT OR THE YOU WANT OF
ANALYSIS IS SOMETHING OTHER THAN
PARTICIPANTS IN THE
PARTICIPANTS, AND BASED ON
CHARACTERISTIC MODULES.
WE ALREADY HAD THOSE EXISTING
FEATURES, SO WE'RE ROLLING THEM
FORWARD IN THE BASELINE
CHARACTERISTICS, AND IN THE
PARTICIPANT FLOW.
IN ORDER TO SHOW A MORE COMPLETE
PICTURE OF THE TRIAL CONDUCT AND
THE TRIAL ANALYSIS.
THERE IS ALSO A FEW OTHER
OPTIONS THAT WE MENTIONED IN THE
PREVIOUS AMBLE FOR THE FINAL
RULE.
THAT ARE BEING MADE AVAILABLE
NOW.
AGAIN, THEY'RE OPTIONAL AND
THEY'RE NOT REQUIRED TO BE USED.
BUT ADDITIONAL OPTIONS FOR
SPECIFYING A NUMBER THAT IS
ACCOUNTS, MEANING THAT WE IN THE
MEASURED TYPE OPTION, IF YOU'RE
FAMILIAR WITH THAT, YOU CAN
BASICALLY INDICATE THAT YOU'RE
REPORTING ACCOUNT --
PARTICIPANTS ARE ACCOUNTED UNITS
INSTEAD OF THE GENERAL OPTION OF
NUMBER.
WE'RE INCORPORATING A NEW OPTION
IN THE ORGAN SYSTEM CLASS,
ADVERSE EVENT REPORTING TO ALLOW
FOR A NEW CATEGORY THAT WAS
INCORPORATED INTO THE MEDRO
SYSTEM, WHICH IS TERMED PRODUCT
ISSUES.
THERE IS ALSO NEW FEATURES ADDED
TO THE PRS, WITH A NEW API
OPTION FOR DOWNLOADING
INFORMATION WHICH SHOULD REALLY
SUPPORT RESPONSIBLE PARTIES AND
INVESTIGATORS AND MANAGING THEIR
PORTFOLIOS, AND BEING ABLE TO DO
A MUCH WIDER RANGE OF THINGS IN
TERMS OF REPORTING.
SO ALL OF THAT WILL BE MADE
AVAILABLE SOON.
I'LL ONLY MENTIONING IN THE
CONTEXT OF THIS PRESENTATION
BECAUSE OF THE DISCUSSION ABOUT
THE OTHER PRS RELEASE RELATED TO
THE FINAL RULE THAT WILL BE
HAPPENING KIND OF MID TO LATE
NOVEMBER.
SO I WANT TO TAKE A STEP BACK,
THEN, AND FOCUS ON SOME OTHER
ASPECTS OF WHICH REQUIREMENTS
APPLY.
AND REALLY FOCUSING ON THE
CLINICAL TRIAL RESULTS
INFORMATION DEADLINES AND WHICH
TRIALS ARE SUBJECT TO WHICH
REQUIREMENTS.
THE FINAL RULE EXPANDS THE
REQUIREMENTS FOR REPORTING TO
APPROVE TRIALS OF UNAPPROVED
PRODUCTS.
IN ORDER TO FIGURE OUT WHICH
TRIALS OF UNAPPROVED PRODUCTS
SUBJECT TO THE STATUTE OR
SUBJECT TO THE FINAL RULE, THERE
REALLY FEED TO BE A COUPLE
DIFFERENT FACTORS TAKEN INTO
ACCOUNT.
ONE IS THE PRIMARY COMPLETION
DATE, AS WE'VE ALREADY
DISCUSSED.
AND THE OTHER IS THE APPROVAL
STATUS OF THE DRUG BIOLOGICAL OR
DEVICE PRODUCT IN THE TRIAL,
ITSELF.
SO I'M GOING TO START WITH
THE -- WITH UNAPPROVED PRODUCTS
AND THE RESULTS REQUIREMENTS.
AGAIN, IF THERE IS AN APPLICABLE
CLINICAL TRIAL, THAT IS NOT
APPROVED LICENSED OR CLEARED FOR
ANY USE BY THE PRIMARY
COMPLETION DATE OF THAT CLINICAL
TRIAL, THEN WE HAVE TO LOOK
TOWARD THOSE EFFECTIVE DATE
REQUIREMENTS TO FIGURE OUT WHICH
REQUIREMENTS APPLY.
SO IF THE PRIMARY COMPLETION
DATE IS BEFORE JANUARY 18, 2017,
OR THE EFFECTIVE DATE, FOR THOSE
TRIALS OF UNAPPROVED PRODUCTS,
MEANIC THAT THE TRIAL INCLUDES
AN UNAPPROVED PRODUCT AS OF THAT
PRIMARY COMPLETION DATE, THEN
THE STATUTORY REQUIREMENTS
APPLY.
RESULTS SUBMISSION IS FOOT
REQUIRED.
IF THE PRIMARY COMPLETION DATE
IS ON OR AFTER JANUARY 18, THEM
THE FINAL RULE REQUIREMENTS
WOULD APPLY, AND RESULT
SUBMISSION WOULD BE REQUIRED FOR
THAT TRIAL OF THE UNAPPROVED
PRODUCT.
THERE ARE SOME DELAYS THAT ARE
POSSIBLE, AND I'LL GO INTO A
LITTLE MORE DETAIL ABOUT THE
MECHANISM OF THAT.
I WANT TO TOUCH BASE BRIEFLY,
THOUGH, ON THE IMPLICATIONS FOR
CERTIFYING INITIAL APPROVAL.
FOR THOSE THAT ARE CURRENTLY
FAMILIAR WITH THE SYSTEM AND THE
OPTIONS THAT ARE AVAILABLE FOR
DESCRIBING AN APPLICABLE
CLINICAL TRIAL, RIGHT NOW IN
CLINICAL TRIALS.GOV, THERE IS
AN OPTION FOR WHAT WE CALL
CERTIFYING INITIAL APPROVAL.
THE WAY THAT THAT IS DEFINED IS
THAT YOU ARE BASICALLY
CERTIFYING THAT THAT PRODUCT
THAT'S BEING STUDIED IN THE
CLINICAL TRIAL IS NOT APPROVED
LICENSED OR CLEARED, AS OF THE
PRIMARY COMPLETION DATE OF THE
STUDY.
AND YOU HAVE THE MECHANISM
BASICALLY TO SUBMIT THIS
CERTIFICATION TO CLINICAL
TRIALS.GOV.
IF YOU HAVE SUBMITTED SUCH A
CERTIFICATION, BASICALLY, AS
DESCRIBED ON THE PREVIOUS SLIDE,
OR IF YOU DIDN'T SUBMIT SUCH A
CERTIFICATION REALLY, RESULTS
ARE NOT REQUIRED TO BE SUBMITTED
EVEN IF THE PRODUCT IS APPROVED
AT A LATER DATE.
WE HAVE MANY TRIALS THAT MAY
HAVE HAD THIS CERTIFICATION IN
PLACE, AND CHOSEN TO SUBMIT
CLINICAL TRIAL RESULTS
INFORMATION ON AN OPTIONAL
BASIS.
ONE OF THE THINGS THAT THE FINAL
RULE PREAMABLE CLARIFIES, THAT
IF RESULTS WERE SUBMITTED
THEY'RE NOT CONSIDERED TO BE A
VOLUNTARY SUBMISSION UNDER THIS
SPECIFIC PROVISION IN 42 CFR
11.60.
WE'LL GET INTO THAT IN A LITTLE
MORE DETAIL IN A FUTURE WEBINAR.
BUT WE JUST WANTED TO MAKE CLEAR
SOME OF THE IMPLICATIONS FOR
FOLKS WHO MAY HAVE ALREADY BEEN
SUBMITTING A CERTIFIED INITIAL
APPROVAL WHICH IS SOMETHING THAT
IS NOT NECESSARILY REQUIRED BY
THE STATUTE ITSELF.
SO NOW TO FOCUS ON THOSE
CLINICAL TRIALS, THOSE
APPLICABLE CLINICAL TRIALS THAT
HAVE A PRIMARY COMPLETION DATE
ON OR AFTER JANUARY 18, 2017, OR
THE EFFECTIVE DATE OF THE FINAL
RILE, THE FINAL RULE ESTABLISHES
WHAT WE'RE CALLING THE STANDARD
SUBMISSION DEADLINE.
BASICALLY, IN GENERAL, RESULTS
INFORMATION MUST BE SUBMITTED NO
LATER THAN ONE YEAR AFTER THE
PRIMARY COMMOTION DATE OF THE
TRIAL.
WITH YOU THE -- COMPLETION DATE
OF THE TRIAL.
BUT THE WAY THE LAW WAS WORDED,
BASICALLY ASKED US TO TAKE INTO
CONSIDERATION SOME OF THE
CERTIFICATION PROVISIONS THAT
WERE DESCRIBED IN THE STATUTE,
ITSELF.
AND SO THE WAY THAT THIS IS
GOING TO BE IMPLEMENTED IN THE
FINAL RULE IS THAT IT IS
POSSIBLE TO DELAY SUBMISSION OF
RESULTS WITH CERTIFICATION, IF
SEEKING INITIAL APPROVAL,
LICENSE OR CLEARANCE.
AND SO BASICALLY, WHAT THAT IS
SAYING IS THAT AGAIN, YOU'RE
ATTESTING TO THE FACT THAT THE
PRODUCT WAS NOT APPROVED,
LICENSED OR CLEARED BY THE FDA
FOR ANY USE BEFORE THAT PRIMARY
COMPLETION DATE AND THAT THERE
IS A GENERAL INTENTION BY THE
SPONSOR TO CONTINUE WITH PRODUCT
DEVELOPMENT, MEANING -- AS KIND
OF A PROXY FOR THE IDEA THAT YOU
WILL BE SEEKING OR INTEND TO
SEEK FDA APPROVAL LICENSURE OR
CLEARANCE.
IN TERMS OF WHAT THE TED LINE IS
FOR WHEN RESULTS INFORMATION
WOULD BE REQUIRED TO BE
SUBMITTED, IF CERTIFICATION IS
IN PLACE, THERE IS A COUPLE
DIFFERENT TRIGS THAT NEED TO BE
LOOKED AT.
IF THAT PRODUCT WERE TO GAIN FDA
APPROVAL LISTENER OR CLEARANCE,
THEN RESULTS WOULD BE DUE WITHIN
30 CALENDAR DAYS OF THAT DATE.
ANOTHER CONSIDERATION IS WHETHER
OR NOT A MARKETING APPLICATION
WAS SUBMITTED BUT WITHDRAWN
WITHOUT RESUBMISSION FOR A
PERIOD OF 210 DAYS.
IF THAT OCCURRED, THEN AGAIN
WITHIN THAT TIME PERIOD, THE
RESULTS MUST BE SUBMITTED 30
CALENDAR DAYS AFTER THAT.
HOWEVER, THE MAIN THING TO
REALLY KEEP IN MIND, THERE IS A
2 YEAR MAXIMUM ON THE
CERTIFICATION PROVISIONS.
SO IF NEITHER OF THOSE THINGS
OCCUR IN THAT EARLIER BULLET IN
TERMS OF FDA APPROVING OR THE
MARKETING APPLICATION BEING
SUBMITTED AND WITHDRAWN, NOT
BEING RE-SUBMITTED, BASICALLY,
AT THE END OF TWO YEARS FROM THE
DATE THAT THAT CERTIFICATION WAS
FILED, THE RESULTS MUST BE
SUBMITTED REGARDLESS OF THE
APPROVAL STATUS AT THAT TIME.
AND SO WHAT THAT MEANS IS THAT
THERE ARE BASICALLY UP TO 3
YEARS FROM THE PRIMARY
COMPLETION DATE, IS THE MAXIMUM
PERIOD OF TIME BY WHICH RESULTS
INFORMATION CAN BE SUBMITTED FOR
THESE TRIALS OF UNAPPROVED
PRODUCTS.
SOMETHING THAT HAS ALREADY BEEN
IN PLACE UNDER THE STATUTE ARE
ADDITIONAL DELAYS FOR CLINICAL
TRIALS, IF THEY ARE EVALUATING A
NEW USE.
SO RIGHT NOW, UNDER THE STANDARD
SUBMISSION DEADLINE RESULTS
WOULD BE DUE WITHIN ONE YEAR OF
THAT PRIMARY COMPLETION DATE.
BUT THERE IS AN OPTION BUILT
BOTH IF I THE STATUTE AND NOW
THE FINAL RULE, ALLOWING FOR
DELAYED SUBMISSION OF RESULTS IF
SEEKING APPROVAL LICENSURE,
CLEARANCE OF A NEW USE.
THERE ARE A COUPLE CONDITIONS
THAT APPLY, AND SO TYPICALLY,
THIS DOESN'T NECESSARILY COVER
ALL RESPONSIBLE PARTIES.
IT'S LIMITED TO A SITUATION IN
WHICH THE MANUFACTURER IS ALWAYS
THE SPONSOR OF THAT APPLICABLE
CLINICAL TRIAL.
AND THE OTHER CONDITION IS THAT
THE SPONSOR HAS FILED OR INTENDS
TO FILE WITHIN ONE YEAR, AN
APPLICATION OR PREMARKET
NOTIFICATION THAT WOULD BE
SEEKING APPROVAL LICENSURE, OR
CLEARANCE, OF THE USE BEING
STUDIED IN THAT CLINICAL TRIAL.
SO ALTHOUGH THERE IS A DELAY
MECHANISM IN PLACE, AGAIN, IT'S
REALLY IMPORTANT TO PAY
ATTENTION TO THE DIFFERENT
CONDITIONS THAT APPLY TO BE
ELIGIBLE FOR USING THIS NEW USE
CERTIFICATION TO DELAY RESULTS
REPORTING.
SIMILARLY, AS TO THE TRIGGERS
FOR WHEN THE DEADLINE FOR
INITIAL -- I'M GOING TO STOP
RIGHT THERE.
WHEN THINKING ABOUT WHEN, THEN,
RESULTS ARE DUE FOR A SITUATION
IN WHICH A CERTIFICATION NEW USE
IS USED, THERE ARE, AGAIN, SOME
OTHER FACTORS THAT COME INTO
PLAY, JUST LIKE WITH THE
CERTIFIED INITIAL APPROVAL, THE
MAXIMUM PERIOD OF TIME BY WHICH
RESULTS INFORMATION SUBMISSION
CAN BE DELAYED IS UP TO 2 -- NOT
LATER THAN TWO YEARS AFTER THE
DATE THAT THE CERTIFICATION WAS
SUBMITTED.
WHICH MEANS NO MORE THAN 3 YEARS
AFTER THE PRIMARY COMPLETION
DATE OF THAT APPLICABLE CLINICAL
TRIAL.
HOWEVER, THERE ARE OTHER
TRIGGERS THAT CAN HAPPEN BEFORE
THAT, THAT WOULD TAKE INTO
ACCOUNT RESULTS REPORTING
EARLIER.
ONE IS IF THE FDA WERE TO
APPROVE THAT APPLICATION FOR
THAT NEW USE, THEN THE RESULTS
WOULD BE DUE WITHIN 30 DAYS OF
THAT APPROVAL.
IF FDA ISSUED A LETTER THAT'S
BASICALLY ENDING THE REGULATORY
REVIEW CYCLE OR THE MARKETING
APPLICATION, OR PREMARKET
NOTIFICATION, WITHDRAWN WITHOUT
RE-SUBMISSION POR THAT TIME
PERIOD OF 210 CALENDAR DAYS,
THEN THE RESULTS WOULD BE TO YOU
WITHIN 30 DAYS OF THE EARLIER OF
ANY OF THOSE EVENTS OCCURRING.
HAVE IS VERY SIMILAR TO WHAT IS
ALREADY IN THE STATUTE, AND
PROBABLY SOMETHING PEOPLE ARE
ALREADY FAMILIAR WITH IN TERMS
OF DEADLINES AND TIMELINES FOR
REPORTING.
ONE OF THE THINGS THAT THE FINAL
RULE DID WAS ALSO CLARIFY THE
CONDITIONS UNDER WHICH INITIAL
CLEARANCE OF A DEVICE PRODUCT
APPLIES.
THESE TWO DIFFERENT TERMS WE
WERE TALKING ABOUT IN THE
CONTEXT OF CERTIFYING INITIAL
APPROVAL, AND CERTIFY A NEW USE
TO ALLOW FOR DELAYS OF RESULTS
SUBMISSION.
FOR THE INITIAL CLEARANCE, WHAT
IT'S TALKING ABOUT IS CLEARANCE
OF A SPECIFIC MANUFACTURER'S
ORIGINAL 510K SUBMISSION FOR A
PARTICULAR DEVICE PRODUCT OF THE
WHEN SPEAK ABOUT A CLEARANCE OF
A NEW USE, IT'S ALSO IN THE
CONTEXT OF THAT SAME
MANUFACTURER'S DEVICE PRODUCT
AND ITS A SUBSEQUENT 510K
SUBMISSION FOR AN ADDITIONAL USE
FOR THAT PARTICULAR DEVICE
PRODUCT MUTUAL THE PREAMBLE OF
THE FINAL RULE ACKNOWLEDGES THE
SITUATION THAT IT MAY NOT ALWAYS
BE KNOWN AT THE TIME THAT
RESULTS SUBMISSION IS DUE,
WHETHER OR NOT IT WOULD BE
CLASSIFIED AS ONE OR THE OTHER
OF THESE, AND BASICALLY, THE
PREAMBLE INDICATES THAT
RESPONSIBLE PARTIES SHOULD USE
THEIR BEST JUDGMENT WITH THE
IDEA BEING THAT AS INFORMATION
IS KNOWN, THE CORRECT ONE WOULD
BE SUBMITTED AS APPROPRIATE.
SO IN TERMS OF WHAT WE INTENDED
TO COVER BROADLY TODAY, IN
TODAY'S WEBINAR, WE WANTED TO
PROVIDE AS HIGH A LEVEL AS
POSSIBLE OVERVIEW OF THE FINAL
RULE AND THAT MAY OR MAY NOT BE
SATISFACTORY FOR SOME, BUT
BECAUSE A LOT OF THE QUESTIONS
THAT WE'VE BEEN GETTING
INITIALLY RELATE TO THE
EFFECTIVE DATE AND THE
COMPLIANCE DATE AND HOW THEY
APPLY TO CURRENT SUBMISSIONS OF
CLINICAL TRIAL REGISTRATION
INFORMATION AND RESULTS
INFORMATION, WE REALLY WANTED TO
TRY TO EXPLAIN IN AS MUCH DETAIL
AS POSSIBLE THE IMPLICATIONES OF
THOSE EFFECTIVE -- IMPLICATIONS
OF THOSE DATES, AND OUR GENERAL
PLANS FOR HOW TO IMPLEMENT THAT
WITHIN THE PRS.
AND TO HELP MANAGE EXPECTATIONS
ABOUT WHEN INFORMATION WILL BE
AVAILABLE.
FOR THE WEBINAR THAT WE'RE GOING
TO CONDUCT NEXT WEEK, ON
OCTOBER 5, IF YOU HAVEN'T
ALREADY REGISTERED, PLEASE DO
SO.
THE LINK FOR REGISTERING IS
AVAILABLE AT THE BOTTOM OF THIS
SLIDE.
IT WILL BE FOCUSING ON THE
CLINICAL TRIAL REGISTRATION
INFORMATION AND UPDATE
REQUIREMENTS AS WELL AS
INFORMATION ABOUT REQUIREMENTS
FOR EXPANDED ACCESS, WHICH FALLS
UNDER THOSE CLINICAL TRIAL
REGISTRATION INFORMATION
REQUIREMENTS.
THEN THE THIRD WEBINAR -- ALSO I
KNOW THAT PEOPLE HAVE BEEN
SUBMITTING A LOT OF QUESTIONS
DURING THE COURSE OF THIS
PARTICULAR WEBINAR.
I'M GETTING VERY LARGE HEAD NODS
AND SMILES PER ERR ONE IN THE
ROOM -- EVERYONE IN THE ROOM.
WE'RE GRATEFUL FOR THOSE
QUESTIONS.
OUR INTENT IS TO BE ABLE TO
FOLLOW UP AS MANY AS WE CAN WIN
THE CONTEXT OF THE NEXT WEBINAR.
AND THE THIRD WEBINAR.
AND IF THOSE AREN'T ABLE TO
ADDRESS THE QUESTIONS DIRECTLY,
TO START ENSURING THAT THE
INFORMATION AVAILABLE ON OUR
WEBSITE CAN AND OR US REFERRING
THOSE QUESTIONS TO OTHER --
WITHIN OUR ORGANIZATION, TO HELP
GET RESPONSES.
SO WE REALLY APPRECIATE THAT YOU
HAVE BEEN SUBMITTING THOSE
QUESTIONS AND THEY WILL BE QUITE
VALUABLE AS WE MOVE FORWARD WITH
IMPLEMENTATION TO MAKE SURE THAT
WE'RE ALL MEETING THE
INFORMATION NEEDS OF THOSE WHO
ARE REQUIRED TO COMPLY WITH THE
RULE.
FOR THE THIRD WEBINAR WE'RE
GOING TO BE FOCUSING ON THE
CLINICAL TRIAL RESULTS
INFORMATION REQUIREMENTS, AS
WELL AS THE UPDATE REQUIREMENTS.
WITHIN THE SECTION OF UPDATES IS
ALSO A SECTION RELATED TO
CORRECTIONS.
THAT'S WHERE A LOT OF OUR
QUALITY CONTROL REVIEW
PROCEDURES ARE ADDRESSED SO
WE'LL GO INTO DETAIL HOW THE
PROCESSES ARE BE CHANGING UNDER
THE FINAL RULE GOING FORWARD AS
WELL AS THE TIMING FOR WHEN
CERTAIN INFORMATION WILL BE
POSTED.
SO AGAIN, PLEASE REGISTER FOR
EACH OF THOSE WEBINARS.
AND WE'LL CONTINUE TO HOPEFULLY
PROVIDE INFORMATION THAT'S
USEFUL.
I WANT TO POINT OUT SOME
ADDITIONAL RESOURCES.
AS DEBRA MENTIONED ON THE DAY
THAT THE FINAL RULE WAS
RELEASED, THE COMPLEMENTARY NIH
POLICY WAS ALSO RELEASED.
AND NIH HAS A NUMBER OF OTHER
CLINICAL TRIAL INITIATIVES THAT
THEY HAVE UNDERTAKEN AND SO THE
NEWS RELEASE ON THE NIH WITNESS
GOES INTO A LITTLE BIT MORE
DETAIL ABOUT THE BROADER CONTEXT
OF THINGS HAPPENING AT NIH.
THAT PAGE, ITSELF, ALSO HAS
LINKS TO THE FINAL RULE TO NIH
POLICY, TO THE NEW ENGLAND
JOURNAL OF MEDICINE ARTICLE THAT
DEBORAH REFERENCED.
AS WELL AS TO ANOTHER DOCUMENT
THAT PROVIDES A SUMMARY OF ALL
OF THE KEY PROVISIONS WITHIN THE
FINAL RULE.
AND A MENTION OF SOME OF THE KEY
THINGS ABOUT THE NIH POLICY.
THE NIH POLICY IS NAMED NIH
POLICY ON THE DISSEMINATION OF
CLINICAL TRIAL INFORMATION.
NIH DID PUBLISH THAT WITHIN THE
FEDERAL REGISTER.
THEY ALSO HAD A COMPLEMENTARY
GUIDE NOTICE.
THAT GUIDE NOTICE ALSO HAS
INFORMATION ON ADDITIONAL
RESOURCES AVAILABLE AT NIH FOR
GETTING INFORMATION ABOUT THE
POLICY.
AND THE GUIDE NOTICE INDICATES
THAT QUESTION ON THE NIH POLICY
SHOULD BE DIRECTED TO THE E-MAIL
ADDRESS HERE.
SO I WOULD ENCOURAGE AND REMIND
EVERYONE IF YOU DO HAVE
QUESTIONS ON THE NIH POLICY,
THAT THOSE ARE BEST DIRECTED TO
THIS E-MAIL ADDRESS HERE RATHER
THAN OUR GENERAL HELP
INFORMATION AT CLINICAL
TRIALS.GOV.
WE WILL DIVERT YOU TO THAT OTHER
E-MAIL ADDRESS, IF YOU HAVE A
SPECIFIC QUESTION ABOUT THE
POLICY.
I ALSO WANT TO HIGHLIGHT SOME
OTHER CLINICAL TRIALS.GOV
RESOURCES.
ON OUR MAIN WEB PAGE, THE PLACE
WHERE WE HAVE HOUSED A LOT OF
INFORMATION ABOUT THE -- ABOUT
THE LAW, AND THE STATUTE, IS
UNDER THE SUBMIT STEADY SECTION.
WE HAVE INFORMATION THERE, BOTH
GENERALLY HOW TO APPLY FOR AN
ACCOUNT WITH US, HOW TO
REGISTER, STUDY, HOW TO SUBMIT
RESULTS INFORMATION, AS WELL AS
BASIC INFORMATION ON WHAT IT IS
THAT THE LAW ITSELF REQUIRES.
AND SO I WOULD ENCOURAGE YOU TO
GO TO THAT WEB RESOURCE TO GET
BASIC INFORMATION ABOUT THE LAW.
WE ALSO HAVE A DOCUMENT POSTED
THERE THAT DESCRIBES SOME OF THE
CHANGES FROM CURRENT PRACTICE IN
THE FINAL RULE.
THAT DOCUMENT INCLUDES KIND OF A
SUMMARY PAGE, AS WELL AS A SIDE
BY SIDE TABLE OF THE DATA
ELEMENTS THAT ARE IN THE FINAL
RULE.
AND A COMPARISON OF WHETHER OR
NOT THEY WERE REQUIRED OR
OPTIONAL IN CLINICAL TRIALS.GOV
PRIOR TO THE FINAL RULE.
WE'VE ALREADY HIGHLIGHTED THIS,
BUT THE ARTICLE IN THE NEW
ENGLAND JOURNAL OF MEDICINE DOES
REALLY PROVIDE A NICE OVERVIEW
OF THE RULE ITSELF, AND MAYBE A
USEFUL TOOL FOR COMMUNICATING
WITH INVESTIGATORS WITHIN YOUR
INSTITUTION.
THE PLACE WHERE YOU REGISTERED
FOR THE WEBINAR WAS THE TRAINING
MATERIALS PAGE WHICH THAT STUDY
SECTION, THAT IS WHERE WE WILL
EVENTUALLY ARCHIVE THE FINAL
RULE WEBINAR SERIES.
IF YOU AREN'T ABLE TO ATTEND ANY
OF THE FUTURE WEBINAR, THEY WILL
BE AVAILABLE FOR ACCESS THERE.
AND THEN FINALLY, WITHIN THE
FINAL RULE ITSELF, WE OFTEN
INDICATED THAT IF PEOPLE -- TO
FIND INFORMATION WITHOUT THE
FINAL RULE, YOU CAN GO TO THIS
WEBSITE, PRS INFO.CLINICAL
TRIALS.GOV.
THIS WAS A WEBSITE WE USED TO
USE BEFORE WE INTEGRATED INTO
THE SYSTEM, SO NOW IT WILL BE
KIND OF A RESOURCE TO FIND OUT
INFORMATION ABOUT FINAL RULE
IMPLEMENTATION.
AND AS WE POST INFORMATION TO
THAT WEB PAGE, WE INTEND TO
CONTINUE TO SEND OUT UPDATES TO
THE FDAAA LISTSERV.
IF YOU GO TO THE WEB LINKS I
HAVE INCLUDED HERE, YOU CAN FIND
INFORMATION ON REGISTERING FOR
THAT LISTSERV.
AND WE'LL HELP PEOPLE IDENTIFY
WHEN THERE ARE NEW RESOURCES
AVAILABLE THAT CAN HELP WITH
THEIR UNDERSTANDING OF THE FINAL
RULE.
FINALLY, IF YOU HAVE QUESTIONS
FOR US, YOU CAN DIRECT THEM TO
REGISTER@CLINICAL TRIALS.GOV.
WE WOULD ENCOURAGE YOU, THERE IS
A LOT OF INFORMATION THAT IS
AVAILABLE IN THE FINAL RULE, AND
TO MAKE SURE THAT YOU'RE USING
ALL THE RESOURCES AVAILABLE TO
YOU BEFORE COMING TO US WITH
QUESTIONS, FIRST, YOU CAN OFTEN
FIND THOSE QUESTIONS THAT YOU
NEED WITHIN THE FINAL RULE
ITSELF, PARTICULARLY IN THE
PREAMBLE, THERE IS A LOT OF
ELABORATION ABOUT EXPECTATIONS
FOR VARIOUS ASPECTS OF THE
REGULATIONS THEMSELVES.
SO I'LL LEAVE IT ON THAT SLIDE,
AND I BELIEVE WE HAVE A FEW
QUESTIONS THAT WE SHOULD BE ABLE
TO ADDRESS NOW.
AND I'M JUST GOING TO TAKE A
SLIGHT PAUSE TO GET THAT
COORDINATED.
[INAUDIBLE]
>> ALL RIGHT.
WE DID HAVE SOME QUESTIONS, KIND
OF BACK TO THE EFFECTIVE DATE
AND THE COMPLIANCE DATE.
AND A LOT OF WHAT YOU MAY REFER
TO AS THE TRANSITION PROVISIONS
IN TERMS OF WHEN THINGS MAY
CROSS THE EFFECTIVE DATE.
ONE OF THE QUESTIONS RELATED TO
WHAT ARE MY OBLIGATIONS FOR
STUDY RECORDS, IF I REGISTER
UNDER THE CURRENT SYSTEM BEFORE
JANUARY 18, SO THAT TRIAL IS
BEING REGISTERED BEFORE THE
EFFECTIVE DATE.
BUT IT TURNS OUT THAT THE STUDY
START DATE DOESN'T OCCUR UNTIL
AFTER THAT EFFECTIVE DATE,
JANUARY 18.
SO THIS IS ONE OF THE SCENARIOS
THAT I TRIED TO GO THROUGH ON
ONE OF THE SLIDES.
IT'S FURTHER EXPLAINED IN THE
PREAMBLE OF THE FINAL RULE THAT
THE EXPECTATION IS THAT IF THAT
TRIAL IS REGISTERED BEFORE THE
EFFECTIVE DATE, BUT THEN THE
STUDY START DATE IS AFTER THAT
EFFECTIVE DATE, SO AFTER
JANUARY, 2017, THE EXPECTATION
IS THAT THAT CLINICAL TRIAL
INFORMATION WOULD BE UPDATED TO
MEET THE FINAL RULE REQUIREMENT.
AND THAT WOULD OCCUR, YOU KNOW,
OVER THAT TIME PERIOD ONCE THOSE
NEW DATA ELEMENTS ARE MADE
AVAILABLE, WHICH WOULD BE IN
THAT TIME PERIOD OF JANUARY TO
APRIL, 2017.
OKAY.
SO ANOTHER QUESTION THAT CAME UP
HAD TO DO WITH PARTIAL RESULTS
INFORMATION.
SO THERE ARE SITUATIONS, AND
THIS RELATED TO THE ELABORATION
OF THE PRIMARY COMPLETE DATE AS
WE DISCUSSED IT A LITTLE BIT.
THAT THIS IS A CASE IN WHICH
THERE IS A STUDY IN WHICH THE
PRIMARY COMPLETION DATE OCCURRED
BEFORE JANUARY 2017.
AND THE RESULTS WERE SUBMITTED
FOR THE PRIMARY OUTCOME MEASURES
FOR THAT CLINICAL TRIAL.
THERE WERE ADDITIONAL SECONDARY
OUTCOME MEASURES THAT WERE BEING
SELECTED ON AN ONGOING BASIS.
SO BASICALLY, THE FINAL DATE OF
DATA COLLECTION FOR THOSE
SECONDARY OUTCOME MEASURES,
ENDED UP BEING THAT THOSE WOULD
BE SUBMITTED AFTER THE EFFECTIVE
DATE OF JANUARY 18, 2017.
SO THE QUESTION WAS, WHEN I
SUBMIT THAT ADDITIONAL RESULTS
INFORMATION WILL I THEN BE
REQUIRED TO UPDATE ALL OF MY
RESULTS INFORMATION TO MEET THE
FINAL RULE REQUIREMENTS?
AND THE ANSWER IS NO, THAT
BASICALLY YOU'RE GOING BACK TO
THE PRIMARY COMPLETION DATE
BEING THE PRIMARY DETERMINANT OF
WHICH INFORMATION NEEDS TO BE
SUBMITTED.
SO IN THIS CASE, IF THE PRIMARY
COMPLETION DATE WAS BEFORE
JANUARY 18, SORT OF REGARDLESS
OF WHEN ANY OTHER INFORMATION
WILL BE SUBMITTED, THE BASIC
THING THAT NEEDS TO HAPPEN IS
JUST FOLLOWING THE FINAL RULE
STATUTORY REQUIREMENTS.
AND THAT PARTIAL RESULTS
INFORMATION WON'T NEED TO BE
UPDATED TO MEET THE FINAL RULE
REQUIREMENTS.
THOSE SECONDARY OUT COME
MEASURES WILL STILL NEED TO BE
SUBMITTED BUT THEY FOLLOW THE
CURRENT REQUIREMENTS UNDER THE
ANOTHER CLARIFICATION ABOUT THE
RESULTS DUE DATE.
THE QUESTION IS ABOUT WHETHER OR
NOT OR NOT IT'S CORRECT
BASICALLY THAT THE WAY THAT THE
REQUIREMENTS WOULD WORK, THAT
THE IMPLICATION IS THAT THE
EARLIEST THAT RESULTS WOULD HAVE
TO FOLLOW THE NEW FINAL RULE
REQUIREMENTS, IS JANUARY 17,
2018.
SO THAT'S A GOOD QUESTION AND
THINKING ABOUT HOW THAT ALL
PLAYS OUT, THAT BASICALLY WITH
THE PRIMARY -- WITH -- BECAUSE
THE EFFECTIVE DATE AND THE FINAL
RULE REQUIREMENTS ARE BASED 0N
THAT PRIMARY COMPLETION DATE FOR
THE APPLICABLE CLINICAL TRIAL,
WHAT HAPPENS IS THE STANDARD
SUBMISSION DEADLINE IS ONE
YEAR/THAT DATE.
LET'S GO WITH FEBRUARY, 2017.
IF THE PRIMARY COMPLETION DATE
WAS FEBRUARY, 2017, THEN WE
WOULD EXPECT THOSE RESULTS TO BE
SUBMITTED BY FEBRUARY, 2018.
IF YOU THINK ABOUT THE TIME
PERIOD OVER WHICH THE RESULTS
REQUIREMENTS WILL BE PHASED IN,
IT'S A LONGER TIMEFRAME BECAUSE
OF THAT ONE YEAR SUBMISSION
>> ANOTHER QUESTION THAT WE HAD
WAS RELATED TO -- THIS HAD TO DO
WITH MORE TECHNICAL THINGS ABOUT
THE PRS, AND WHETHER OR NOT IT
WILL BE POSSIBLE TO USE ANY OF
THE NEW DATA ELEMENTS FOR AN
OLDER STUDY THAT MAY NOT BE
SUBJECT TO THOSE FINAL RULE
REQUIREMENTS.
A CURRENT PLAN FOR
IMPLEMENTATION, THOSE NEW DATA
ELEMENTS WOULD BE AVAILABLE FOR
ANY CLINICAL TRIAL THAT'S BEING
REGISTERED.
WHAT THE SYSTEM WILL, ITSELF,
ENFORCE, THOUGH, WILL BE MORE
BASED UPON THOSE EFFECTIVE DATE
COMPLIANCE DATE ISSUES.
SO IT WOULD TECHNICALLY BE
POSSIBLE TO PROVIDE SOME OF
THOSE NEW DATA ELEMENTS, BUT IF
THOSE DATA EMMENTS ARE PROVIDED
THEY MUST MEET THE GENERAL
VALIDATION RULES OF THE SYSTEM
AND ANY REVIEW CRITERIA THAT
MIGHT BE ASSOCIATED WITH THE
SUBMISSION OF THAT INFORMATION.
THERE IS A QUESTION ABOUT PHASE
1 STUDIES.
AND HOW THAT RELATES TO THE
FINAL RULE.
ONE OF THE THINGS THAT DEBRA --
[TECHNICAL DIFFICULTIES] -- ONE
OF THE THINGS IS THE DEFINITION
OF AAPPLICABLE TRIAL.
BOTH IN THE STATUTE AND FINAL
RULE, PHASE I STUDIES ARE
EXCLUDED FROM THE DEFINITION OF
AN APPLICABLE CLINICAL TRIAL.
SO GENERALLY SPEAKING, THOSE ARE
NOT COVERED BY THE FINAL RULE.
ONE OF THE THINGS I ALLUDED TO
IN -- ALLUDED TO IN ANOTHER
SLIDE WAS RELATED TO VOLUNTARY
SUBMISSIONS, AND WE'RE NOT GOING
TO BE COVERING THAT NOW BUT I
RECOMMEND READING SECTION 1160
WHICH TALKS ABOUT VOLUNTARY
SUBMISSIONS AND WE WILL COVER
THAT IN MORE DETAIL IN A
SUBSEQUENT WEBINAR.
GENERALLY, PHASE I TRIALS ARE
NOT CONSIDERED TO BE APPLICABLE
CLINICAL TRIAL.
LOOKS LIKE WE'VE REACHED THE END
OF QUESTIONS THAT WE'RE GOING TO
BE ABLE TO ADDRESS HERE.
AS I MENTIONED, WE HAVE
CATALOGED ALL THE ADDITIONAL
QUESTIONS THAT HAVE COME IN.
AND WE WILL ATTEMPT TO ADDRESS
AS MANY OF THOSE AS WE CAN ON
THE SUBSEQUENT WEBINAR.
I REMIND YOU OF ALL THE
RESOURCES THAT ARE AVAILABLE,
THEY'RE UP ON THE SCREEN RIGHT
NOW.
AND WE WILL PLAN TO MAKE THIS
AVAILABLE AFTER WARD.
SO WE REALLY THANK YOU FOR
JOINING US TODAY.
WE KNOW THAT THE RULE HAS A LOT
CONTAINED WITHIN IT, AND THERE
IS A LOT TO LEARN.
SO WE'RE LOOKING FORWARD TO
CONTINUING THIS JOURNEY IN THE
NEXT WEBINAR, AS WELL AS THROUGH
OUR OTHER MATERIALS.