electronic data submission in japan -current status and future - …€¦ · preparation for the...

ElectronicDataSubmissioninJapan

- CurrentStatusandfuture

YukiAndo,PhDSeniorScientistforBiostatisticsOfficeofAdvancedEvaluationwithElectronicDataPharmaceuticalsandMedicalDevicesAgency(PMDA)

Outline

• Currentstatusofe-datasubmissioninJapan– Timeline– Preparationfortheendofthetransitionalperiod

• Utilizationofaccumulateddatainthefuture

2018/07/10 CDISCJapanInterchange2018 2

Outline




Accumulationandutilizationofdata


Submissionofelectronicclinicalstudydatahas

startedsinceOct1st 2016!

Timelineforimplementationofe-datasubmission


Task J-FY2014

J-FY2015

J-FY2016

J-FY2017

J-FY2018

J-FY2019

J-FY2020

Guidanceandrelateddocuments

Review

Consultationfore-studydatasubmissionSystem

Development

2014Pilot

2015Pilot

Pilot

SystemDevelopment/Pilotfordatasubmission

Issuanceof“BasicPrinciples”

3.5yearsofTransitionalperiod

FAQ

Briefing/Workshop

Eng.

Mar31

Endofthetransitionalperiod

RegularUpdate

Preparationfortheendoftransitionalperiod

(Revisionofthenotifications,etc)

Today

WS

Oct1

PortalSite Open

NewConsultationframework

Issuanceof“Notificationontheconsultationfortheclinicale-datasubmission”

Issuanceof“NotificationonPracticalOperations”

Issuanceof“TechnicalConformanceGuide”

DataStandardsCatalog

PMDAValidationRules

Initiationofe-studydatasubmission

WS WS

Q&A

Notifications,Guide,FAQs,andStandardsCatalog

• BasicPrinciplesonElectronicSubmissionofStudyDataforNewDrugApplications(Jun20,2014)andQ&A– ThefirstofficialannouncementthatMHLW/PMDAwillrequireCDISC-

standardizedstudydataforNDA.• NotificationonPracticalOperationsofElectronicStudyDataSubmissions

(Apr27,2015)andQ&A– Practicalissuesone-Studydatasubmission– Q&AwasupdatedonMay17,2018

• TechnicalConformanceGuideonElectronicStudyDataSubmissions(LatestupdateonMay17,2018)– Technicaldetailsone-Studydatasubmission

• FAQwebsite(LatestupdateonMar7,2018)– Supplementalexplanationsbasedonthefrequentlyaskedquestionsatthe

meetingwithsponsorsandthecommentstothenotificationsandtheguide• PMDADataStandardsCatalog(LatestupdateonMar3,2017)

– ListofacceptableversionsofDataExchangeStandardsandTerminologyStandards


Timelineforimplementationofe-datasubmission


Task J-FY2014

J-FY2015

J-FY2016

J-FY2017

J-FY2018

J-FY2019

J-FY2020

Guidanceandrelateddocuments

Review

Consultationfore-studydatasubmissionSystem

Development

2014Pilot

2015Pilot

Pilot

SystemDevelopment/Pilotfordatasubmission

Issuanceof“BasicPrinciples”

3.5yearsofTransitionalperiod

FAQ

Briefing/Workshop

Eng.

Mar31

Endofthetransitionalperiod

RegularUpdate


(Revisionofthenotifications,etc)

Today

WS

Oct1

PortalSite Open

NewConsultationframework

Issuanceof“Notificationontheconsultationfortheclinicale-datasubmission”

Issuanceof“NotificationonPracticalOperations”

Issuanceof“TechnicalConformanceGuide”

DataStandardsCatalog

PMDAValidationRules

Initiationofe-studydatasubmission

WS WS

Q&A

Transitionalperiodwillbeended…

• ThetransitionalperiodwillbeendedonMarch31,2020.– Duringthetransitionalperiod,applicantscansubmitthedataofatleastoneclinicaltrialincludedintheirclinicaldatapackages.

– Aftertheperiod,applicantsneedtosubmitthedataofalltherequestedclinicaltrials.


2014 2015 2016 2017 2018 2019 2020 2021 2022 2023-

Oct1 Mar31

42monthsofthetransitionalperiod

NotificationonPracticalOperationsofElectronicStudyDataSubmissionspublishedon

Apr27,2015

Ifsubmissiondateisafterthisdate,applicantsneedtosubmitthedataofalltherequestedclinicaltrials.

Apr27


Weareconsideringrevisionofnotificationsandclarification,forexample,

• RevisionofnotificationswithconsideringsituationwhenapplicantscannotuseElectronicSubmissionGateway

• Clarificationofrelationshipbetween– Electronicstudydatasubmission– SubmissionofeCTD– UseofElectronicSubmissionGateway

• Clarificationoftimingofdatasubmissionforspecialsituation– Ex.Newdrugapplicationsforanti-HIVdrugs


Wewillproceedtheprojectwithcontinualdiscussionwithindustryforthesmoothtransitiontothenextphase.

SituationswhenapplicantscannotuseESG

• OriginallywehadexpectedthatapplicantscouldsubmittheelectronicdatatothePMDAwindow(notviatheESG)onlyincaseofsystemfailure.

• Basedontheexperiences,wewillalsoconsiderothersituationsasunavoidablecircumstances.

• TherevisionsonMay17,2018– “NotificationonPracticalOperations”Q&A4– “TechnicalConformanceGuide”3.7– FAQ3-24


DatasubmissionandeCTD

• Therelationshipbetweensubmissionofstudydata,submissionofeCTD,andusingEDG,foreachsubmissioncategorywasambiguous.

• Therevisionof“NotificationonPracticalOperations”Q&AonMay17,2018– NewQ&As16to18– AtableclearlyshowingtherequirementofCTD

oreCTD submissionandavailabilityofESG,foreachsubmissioncategory(e.g.Drugwithnewactiveingredients)wasadded.


Timingofdatasubmissionforspecialsituations

• Therearesomesituationswhereactualreviewismainlyconductedbeforeofficialsubmissionofnewdrugsapplication.– Newdrugapplicationsusing“Sakigake”designationscheme

– Priorassessmentconsultation– DrugsforHIVinfection

• Commonbasicconceptforthesituations:– Applicantscanvoluntarilysubmitstudydatabefore

thesubmissionofnewdrugapplication.– Officialstudydatasubmissionisrequestedatthe

timeofsubmissionofnewdrugapplication.2018/07/10 CDISCJapanInterchange2018 12

OthertopicsofCDISCrelatedissues

• ExceptionalcaseofSIunit– IntherevisionofFAQonMay17,2018,adescriptionaboutmmHgwasaddedtoFAQ4-8.“TheuseofSIunitsisrequiredforallvariablesandparametersoftestresultstobestoredintheFindingsclassdomainoftheSDTMdataset,aslongasSIunitsareapplicable.However,incasetestresults(e.g.bloodpressure)arecollectedusingmmHgasconventionalunit,onlythetestresultsinmmHgcanbestoredinSDTMdatasetswithoutconvertingthemtotheresultsinSIunit.”


OthertopicsofCDISCrelatedissues

• CDISCValidationinPMDA– WeplantoupdateourCDISCvalidationsoftwareandPMDA

validationrulesinthenearfuture.– Weareconsideringsmoothtransitiontothenewversionand

thedetailedinformationwillbeprovidedinadvance.• Useofnon-clinicaldata (SEND)

– Wecontinuetodiscusshowwecanefficientlyusenon-clinicalstudydatainourreviewprocess.

– Submissiontiming willbeoneofthe topics in the discussion.• TherapeuticAreaStandards

– WecontinuetoworkforincorporatingmedicalpracticesinJapantoTherapeuticAreaUserGuidesincollaborationwithacademicsocietiesandindustry,mainlyinthePublicReviewperiod.

– WebasicallyacceptthatapplicantsimplementthepublishedTAUGs.


Outline




RegulatoryScienceCenterestablishedonApr1


Organizationchange

• Nowweareworkingforelectronicdatasubmissioninthenewoffice,“OfficeofAdvancedEvaluationwithElectronicData”,apartoftheRegulatoryScienceCenter.


www.pmda.go.jp/files/000223757.pdf

RegulatoryScienceCenter

Accumulationandutilizationofdata


Trendsofutilizationofnovelmethodologies


https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm505485.htm

http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000319.jsp

Internationaldiscussionaboutdatasources


AsthefirststepofGCPRenovation,theExpertWorkingGrouphasstartedthediscussionofrevisionofE8Guidance

ReflectionPaperofGCPRenovation

ICH:TheInternationalCouncilforHarmonisationICHE8:GeneralconsiderationsforclinicaltrialsICHE6:GoodClinicalPractice

www.ich.org/products/gcp-renovation.html

Utilizationofstudydatainthefuture


Utilizationofstudydatafornewdrugreview•- Improvementofpredictabilityofefficacyandsafety•- ReviewingM&Sresults•- Reviewingnovelevaluationmethods•- Swiftandeffectivedecision-making

Utilizationofaccumulatedstudydata•- Informationfromcross-productanalysis•- ActiveuseofM&S•- Evaluationofinnovativeanalysismethodsbasedontheaccumulateddata•- Experiencesofmeta-analyticapproach

Efficientnewdrugdevelopment•- Useofconsultationmeetingbasedonthecross-productinformationbyPMDA•- ActiveuseofM&S•- Useofinnovativeandappropriatemethodsforthepurpose

- Consultationbasedonthecross-productinformation- Guidancefortherapeuticareas- Issuanceofpointstoconsidersformethodology

Submissionofstandardizedstudydata

- Dataaccumulation- Experiencesofdataevaluation

Useofvariousdatasourcesinthefuture- Importanceofstudyquality,dataquality,anddatastandardization- Innovativemethodsforanalyzingdatafromvariousdatasources

- Consultation/guidanceaboutinnovativeanalysismethods- Contributiontodatastandardization

electronic data submission in japan -current status and future - …€¦ · preparation for the...

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