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GlobalDataSharingandTransparencyIni7a7ves
BarbaraE.Bierer,[email protected] May3,2016
F494:TheNewWorldofTransparencyforClinicalTrialResults
Disclaimer:
• Theopinionscontainedhereinarethoseoftheauthorsandarenotintendedtorepresenttheposi7onofBrighamandWomen'sHospitalorHarvardUniversity.
• TheMRCTCenterissupportedbyvoluntarycontribu7onsfromfounda7ons,corpora7ons,interna7onalorganiza7ons,academicins7tu7onsandgovernmenten77es(seewww.MRCTCenter.org)andwellasbygrants.
• WearecommiOedtoautonomyinourresearchandtotransparencyinourrela7onships.TheMRCTCenter—anditsdirectors—retainresponsibilityandfinalcontrolofthecontentofanyproducts,resultsanddeliverables.
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Engagediversestakeholderstodefineemergingissuesinglobalclinicaltrialsandtocreateandimplementethical,ac7onable,andprac7calsolu7ons.
OurMission
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MRCTCurrentProjectStatus
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TrainingandCompetencyProtocolEthicsReturnofAggregateResultsTrainingandCompetenciesGlobalRegulatoryEngagementEngagemeDataSharingandTransparencyPost-TrialResponsibili7es
IndividualReturnofResults
4
Purpose-drivendatasharingwillenhancescien7ficdiscoveryandpublictrust
• Benefitsaresubstan7al(e.g.):– Eliminateduplica7vetrials– EvaluatecommonAEsbycompoundclassorsubpopula7on– Iden7fysurrogateendpoints– Enhancecorrela7veandexplanatoryscience
• Butbenefitsonlyrealizedif:– Risksareminimized,withaOen7ontopar7cipantprivacy
• AOen7ontoinformedconsentandrespectforconsent• Dataanonymiza7on
– Dataareinteroperable,datasetscanbepooled• Datastandardsareavailable,oralterna7vemethodology• Metadataisshared• Real-7meanaly7csareavailable
– State-of-the-artsecurityisinplace
2012-2015MajorMilestonesinClinicalTrialDataSharing:Plaaorms
• GSKandtheClinicalStudyDataRequest(CSDR)plaaorm,amul7-sponsorrequestsitewheredataareusedinasecureenvironment
• J&J-YODAprojectthroughwhichYalepartnerswithJ&JandMedtronic
• Pfizer’sIntegratedSystemforPfizerPIIni7atedResearch(INSPIIRE)Portal
• DukeClinicalResearchIns7tute–BristolMyersSquibbStrategicIni7a7ve(SOAR),whichsupportsopenaccesstoclinicaltrialsdata
• NIHBioLINCCdatarepositoryandothers
• FDA-ProjectDataSphere(cancercompara7vedata)andHigh-PerformanceIntegratedVirtualEnvironment(HIVE),aprivate,cloud-basedenvironmentthatcomprisesdatastoragelibrary,algorithmsforanalysis,andcomputa7onalcapacity
HoweverthesearenotinteroperablenorarethesesystemsintegratedFewvoluntaryacademicorbiotechdatageneratorparWcipants
AccesstodocumentsRequestsforinforma7on
7
• Policy published November 2010
• ‘Outputs’ table to guide
implementation
• Change in access policy had big
impact on Agency operations
• Average 450-500 requests for
information each month
• But information only goes to the requester and
although they may share, it is less visible
Clinical trial transparency
8
EUClinicalTrialsRegister
ProacWvepublicaWonofclinicalstudyreports–Policy70
ClinicalTrialRegulaWonandEUPortalandDatabase–publicinformaWonclinicaltrialsauthorizedinEU
ICJMEdatasharingproposal,26Jan2016
• TheICMJEdefinesaclinicaltrialasanyresearchprojectthatprospec7velyassignspeopleoragroupofpeopletoaninterven7on,withorwithoutconcurrentcomparisonorcontrolgroups,tostudythecause-and-effectrela7onshipbetweenahealth-relatedinterven7onandahealthoutcome.
• “Asacondi7onofconsidera7onforpublica7onofaclinicaltrialreportinourmemberjournals,theICMJEproposestorequireauthorstosharewithothersthedeiden8fiedindividual-pa8entdata(IPD)underlyingtheresultspresentedinthear8cle(includingtables,figures,andappendicesorsupplementarymaterial)nolaterthan6monthsaEerpublica8on.Thedataunder-lyingtheresultsaredefinedastheIPDrequiredtore-producethear7cle'sfindings,includingnecessarymetadata.Thisrequirementwillgointoeffectforclinicaltrialsthatbegintoenrollpar7cipantsbeginning1yearakertheICMJEadoptsitsdata-sharingrequirements.”*
• Anyonecanprovidefeed-backatwww.icmje.orgby18April2016.
hOp://www.icmje.org/news-and-editorials/M15-2928-PAP.pdf
MRCTCenter’sRoleasaNeutralConvener
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Nonprofits
Industry
Pa7entsand
Pa7entOrg
Academia
Gov’t
Journaleditors
Tocreateimplementablesolu1onsfordatasharing
DataSharing:CommonICFandDUA
TEMPLATE ICF LANGUAGE FOR DATA SHARING* DATA USE AGREEMENT TEMPLATE
*Panelat2:30todaywilldiscussissuesininformedconsent
DataSharingConference:ConsensusofFutureVisionMarch30-31,2015
• 70representa7vesofpharma,biotech,pa7ent/pa7entadvocates,founda7ons,academics,journaleditorsandothers:
• Consensusonfuturestrategicvision:1. Expecta7onsandprac7cesofregistra7onandresultsrepor7ngofall
clinicaltrialswouldberegularizedamongindustryandacademia;
2. Greateraccesstopar7cipant-levelclinicaltrialdatacouldbefacilitated;
3. Researcherswouldbeabletoaccessandcombinedataacrossvariousplaaormsandsponsors,tomul7plyopportuni7esfordataanalysis;and
4. Researchpar7cipantprivacymustbesafeguarded,throughITdesignaswellascondi7onsimposedondatausers.
6/6/16 13SponsoredbytheMRCTCenter,LauraandJohn
ArnoldFounda8onandWellcomeTrust
March2015DataSharingConference:FutureVision
• OrganizaWonalstructureandGovernance-Acoordina7ng,centralized,interna7onal,not-for-profitorganiza7onwithaccountability;
• Acentralizedandsingleportal-Acentraluserinterfacewitharobustsearchenginefunc7onality,includinginforma7onontrialsaroundtheworld;
• Datarequirements–Datastandards,defini7on,dataontologyandmetadatatoallowforandenabletheintegra7onofdifferingdatasetsforanalysis;
• Sharedorcommonservices–Efficientsharedorcommonservicesacrossdatagenerators/sponsors(e.g.policysesng,datade-iden7fica7on,criteriaforindependentreviewpaneldecisionsorreliance,andsta7s7calservices);and
• Flexibility–Dataplaaormaccommoda7ngdifferingexpecta7onsandneeds:abilitytohostdata,accessdatafromthirdpartyhosts,ordownloaddataiffreelyavailable.Abilitytou7lizemiddlewaretocommunicatewithanalysisapplica7ons.Analy7cflexibilitynecessary.
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ClinicalTrialDataSharingandTransparencyProject
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ClinicalTrialData
StudyParWcipants
ResearchersPublic
The MRCT Center, the Wellcome Trust, and Arnold Foundation and collaborators have spearheaded a major initiative related to sharing of clinical trials data, to enable stakeholders to comply with ICMJE proposed requirements, pending EU regulations, and IOM guidelines inter alia on clinical trial data sharing. Our goal is to ensure a collaborative, unified approach to a common platform and portal. We convened stakeholders from the US and Europe and beyond on March 21-22, 2016 at the Wellcome Trust to vet the vision of a global, federated portal of all data sharing sites from industry, academia and others with the capacity to host data and render such data interoperable.
• InStrategy:Wehavelaunched3integratedworkinggroupstodeveloporganiza7onalblueprintforthesuggestednot-for-profiten7ty:
16
MRCTGovernance
WorkingGroup
Informa7onTechnology(IT)WorkingGroup
BusinessModels
WorkingGroup
Definepurpose,plan,governanceandscopeofnewen7ty
Developglobal-levelITplaaormblueprint
Developsustainablebusinessmodel
DataSharing:StrategyPhase
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TheMRCTCenter’sDataSharingWorkgroupMembers
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GovernanceWorkStream ITWorkStream BusinessModelsWorkStream
Co-Chairs:MRCTCenterWellcomeTrustArnoldFoundaWon
Co-Chairs:IdaSim(UCSF)BarbaraBierer(MRCT)
Co-Chairs:WellcomeTrustMRCTCenter
TeamMembers:MarkBarnes(MRCTCenter)BarbaraBierer(MRCTCenter)StuartBuck(ArnoldFoundaWon)MarlaJoBrickman(Pfizer)NinaHill(Pfizer)RebeccaLi(MRCTCenter)NickLingler(DeloiceConsulWng)Jus7nMcCarthy(Pfizer)HeatherMarino(MRCTCenter)SandraMorris(Johnson&Johnson)JenniferO’Callaghan(WellcomeTrust)NicolaPerrin(WellcomeTrust)PaulSeligman(Amgen)IdaSim(UCSF)JessicaScoO(GlaxoSmithKlein)CatrinTudurSmith(UniversityofLiverpool)NatalieZaidman(Pfizer)
TeamMembers:GeorgeAlter(UofMichigan)MuntherBaara(Pfizer)BarbaraBierer(MRCTCenter)KrisBolt(MRCTCenter)BrianBot(SageBionetworks)AnneClaiborne(IOM)KhaledElEmam(UofOcawa)GhassanKaram(WHO)MichaelKhan(UofColorado)SeanKhozin(FDA)RebeccaKush(CDISC)RebeccaLi(MRCTCenter)GeneLichtman(HCRI)MichelleMancher(IOM)HeatherMarino(MRCTCenter)ChrisMavergames(Cochrane)EricPerakslis(Takeda)FrankRockhold(GSK)
TeamMembers:BarbaraBierer(MRCTCenter)PatrickCullinan(Takeda)RebeccaLi(MRCTCenter)PeterLyons(Deloice)HeatherMarino(MRCTCenter)NicolaPerrin(WellcomeTrust)RohinRajan(Deloice)
17
Vision
Toadvancehumanhealththroughclinicaltrialsdatasharing,therebyrespec7ngandhonoringthecontribu7onsofsponsors,funders,inves7gatorsand,mostessen7ally,clinicaltrialpar7cipants
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MissionPromote,coordinate,andfacilitateclinicalresearchdatasharingthroughthecreaWonandimplementaWonofasustainableglobaldata-sharingenterprisethatwill:
• Protectstudypar7cipants’privacyandrespectthelegi7mateinterestsofdatagenerators,fundersandsponsors
• Encompassthefullbreadthofclinicaltrialsfundedandconductedbyacademia,government,industryandothers
• Respectandbridgetoorincorporateexis7ngdatasharingplaaorms
• Providethecapabilitytohostandanalyzedata,aswellastoenablediscoveryofdataonexternalorgeneratorplaaorms.
• Interactwithandcomplementcurrentregistries,resultsrepor7ngplaaorms,andregulatoryini7a7ves
• Provideanindependentreviewprocessfordatarequests,whererequired
• Developglobal,fairdatasharingpoliciesandprac7ces6/6/16 19
Scope
Thisdatasharingini7a7vewillfunc7onasaplaaormforthesharingofclinicaltrialsdatabyhos7ngdataforstakeholdersthatmaylackthenecessaryresourcestodosoandbycoordina7ngandintegra7ngexis7ngini7a7ves,asappropriate.Phasedapproach:• PhaseIlaunch,withdefini7onofminimumviableproduct(MVP)• PhaseIIandbeyond:acquisi7onanddevelopmentofaddi7onal
func7onali7es
FocusonIPD
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• Vivli,adaptedfromtheGreekwordforlibrary,‘vivliothiki,”andtheLa7nroot“viv,”orlife.
• WehopeVivli“thelibraryoflife,”willevokecoopera7on,collabora7onandadetermina7ontorespectthealtruismofclinicaltrialpar7cipantsworldwideforthebenefitofmedicineandpublichealth.
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Thedatasharingorganiza7on:iden7tyandbrand
Vivlitoestablishresourcesandexper7setopromotetransparency,standardsandequity
• Abilitytohostdataandaggregatedata
• Dynamicanaly7ctools
• Commontemplatesandresources:– Publiceduca7onandtools– Informedconsenttemplates,op7mizedforcountryandpurpose
– Datacontributoragreements
– Enforceabledatarequesteranddatauseagreements,specificforpurpose,thatprohibitre-iden7fica7oninteralia
• Independentreviewpanels(orrelianceagreements)fordatathataresubjecttointermediaryreview
• Datastandardsanddatacura7on
• Risk-baseddataanonymiza7on
• Developmentofstandardsandframeworkstointegratedataplaaorms
• State-of-the-artsecurityprovisionstominimizerisk6/6/16 22
ProposedOrganiza7onalStructureforNonProfitVivli
BoardofDirectors
Finance&AuditCommiOees
AdvisoryTechnicalCommiOee
ExternalAdvisoryCommiOee
President/ExecDirector
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24
ApproachtotheWork
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UseCaseDocuments–Definescope,u7lityandfeaturesetofITplaaormincluding:
• DataAnalysisUseCases• Outlinehowdatauserswillinteract
withtheplaaormtofind,request,andanalyzedata
• Analyzingpublishedandunpublisheddatafromstudies
• DataSubmissionUseCases• Outlineshowdatageneratorswill
depositdataintheplaaorm• Addressesanonymiza7onanddata
standardiza7onissues
MRCTCenterITWorkgroup
PlaeormUseCases
DataSharingPlaaorm
5Essen7alDomainsforPlaaorm
PlaeormTopics
IPDIntakeandCuraWon Mustbeabletointakeandcuratedatawithflexibilitytoscaletonewtypesofdata
CatalogMetadataandDatasetIdenWfiers
Musthaveaccuratestructuredmetadataanduniqueiden7fiersforeachdataset,toenableprecisegranularsearching
ReviewProcessforRequests Mustbeabletoaccommodatedifferentreviewrequirementsandprocesses
AnonymizaWonandDUAs Cri7caltomaintainpar7cipantprivacy
AnalysisToolsandWorkspace Musthaveasecureanalysisspace,enablingasmuchcross-datasetaggregra7onaspossible,withtheflexibilitytodevelopaccommoda7onofmanytypesoftools
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Overviewofplaaormdatasharingscenarios
26
VivliPlaeorm
CatalogMetadata
Non-IPDe.g.,protocol,SAP,
IndividualPaWentData(IPD)
Analy7cEnvironmentandTools
SAS R etc.
Query,Request,andAnaly7csUserInterface
IPD
IPD Non-IPD
R
ScenarioA
ScenarioB
Non-IPD
SAS
PossibilitytoaggregatedatasetsacrossSAS
environments
DataRequester
Userauthen7ca7onDatauseagreement
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250trials
Sponsorno8fiesplaOormof#of
par8cipa8ngtrials
Plaaormcurates
metadataforthe250trials
IPDSHARED
SearchabletermsmatchavailableIPD
Reviewprocess
PlaaormPar7cipa7ngTrials(e.gSponsors)
• NoIRPreview• Plaaormreview• Datageneratorreview
PlaaormPar7cipa7ngTrials(e.g.Publica7on-based)
• Publica7onpreparedforsubmission
SubmissiontoJournal
• DataPackagePreparedforPlaaorm(anonymized)
SubmissiontoPlaaorm
• IPD/DataPackageandPublica7onheldinEmbargo
Ar7clePublished&Dataavailableonplaaorm
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WhyVivliisNeeded
• Datahos7ngcapacity• Analy7cfunc7onality/value• Economiesofscale
– Technical– Policy– Opera7onal
• Lowercostssecondarytoeconomiesofscale
Ø Buildingupon,leveraging,andpartneringwithexis7ngplaaormsandcrea7nganewonlythatwhichisessen7al
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Connec8ngcurrentclinicaltrialplaOormsHos8ngtrialsthatdonotcurrentlyhaveahomeOfferingaddi8onalservices- Advancedsearchcapabili8es- Anonymiza8on- Sta8s8calanalysistools
-
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Anewglobalplaeormfordatasharing