利用真实世界的证据支持肿瘤靶向治疗（英）

WhitePaper LeveragingRealWorldEvidencetoSupportOncologyTargetedTherapies Tableofcontents Executivesummary3 Introduction:RealWorldEvidence(RWE)forprecisiononcology4 Challenge:RWEwithnovelbiomarkers5 Solution1:Accesstolinkedbiomarker&clinicaldata6 Solution2:Retrospectivetestingofarchivaltissues7 Solution3:EnablingAI-Poweredprecisiononcology8 Conclusion9 Abouttheauthor9 References10 Executivesummary IQVIA'sOncologyEvidenceNetwork(OEN)collaborateswithacademichospitalsandcancercenterstoproducesignificantreal-worldevidencestudiesinoncology.Withmorethan30partners,OENhasenabledmanyRWEstudiesacrossabroadrangeofoncologyindications,includingnaturalhistory,comparativeeffectiveness,externalcomparator,andprecisiononcologystudies. OurwhitepaperprovidesanoverviewofacomprehensivesolutionforgeneratingRealWorldEvidence(RWE)inthefieldofprecisiononcology. OncologytargetedtherapiescansignificantlybenefitfromRWEprograms,speedingupclinicaldevelopment,supportingmarketaccess,anddrivingmarketpenetration. However,theadoptionofnovelbiomarkersinclinicalpracticeisslow.This,combinedwiththevariabilityintestingpracticesandthelow prevalenceofsomenovelbiomarkers,makesRWE programsunpredictable,costlyandevenunfeasiblein certaincases. OurteamhasdevelopedacomprehensivesolutiontoincreasethefeasibilityandmaximizethevalueofRWEprogramsfortargetedcancertherapies,including: •Accesstolinkedbiomarker&clinicaldata:BypartneringwithleadingoncologycentersinEurope,thissolutioncombinesrichbiomarker datawithclinicalElectronicMedicalRecord(EMR)data.ItenablesvarioustypesofRWEstudiesandenhancesefficientsiteselection,expeditingevidencegenerationandmarketaccess. •Retrospectivetestingofarchivaltissues:Collaborationwithbiobanksandpathologylabsallowsforretrospectivetestingofarchivaltissuesamples,broadeningourunderstandingofnovelandunder-testedbiomarkers.Thisefficientandcost-effectiveapproachspeedsupprecisiononcologystudies. •EnablingAI-Poweredprecisiononcology:Thissolutionstreamlinesaccesstoimagingandpathologydataforpharmaceuticalpartners. Whetherfordetectingoncologybiomarkersfrompathologyimagesordiscoveringradiomics-basedbiomarkers,thesedatamodalitiesarekeyforleveragingmachine-learningtechnologies. OurdedicatedsolutionenablesustoruneffectiveRWEprogramsfortargetedcancertherapies,evenfornovelandunder-testedbiomarkers,andhelpsourpharmaceuticalpartnersgetthemostvaluefromtheseprograms. Introduction:RealWorldEvidence(RWE)forprecisiononcology Targetedtherapiesareinstrumentalinrevolutionizingcancertreatment,astheyprovidepersonalizedcaretoeachpatient.Productsthatnecessitatebiomarkertestingaccountforapproximately60%oftheoncologymarketshare1.Remarkably,thissegmentisexperiencingthehighestgrowthratewithinoncology,boastingarobustcompoundannualgrowthrate(CAGR)of21%1.Anexaminationoftheoncologypipelinesuggeststhatthistrendisexpectedtopersistinthefuture. Thesetargetedtherapiesinherentlyfaceauniquesetofchallengeswhenitcomestogeneratingclinicalevidenceandmarketentry.ThisiswhereRealWorldData(RWD)playsacrucialrole.Drawingfromourexperienceinassistingleadingpharmaceuticalcompanies,RWDoffersnumerousbenefitsinthedevelopmentandcommercializationoftargetedoncologytherapies. Thesebenefitsinclude Acceleratingclinicaldevelopmentbyutilizingrealworldcontrolarms:LeveragingRWDasexternalcontrolarmscanacceleratepivotalclinicalstudies.Thisisparticularlybeneficialfortargetedtherapies,wherepatientnumbersareinherentlylow.Intheperiodof2019-2021,85%ofFDAsubmissions(NDAsandBLAs)madeuseofRWDfortherapeuticcontext,safety,effectiveness,oracombinationthereof2.Similarly,RWDservedasanexternalcomparatorforclinicalefficacyverificationin17%ofEMAsubmissionsfrom2016to20213. SupportingmarketaccessbygeneratingrobustRealWorldEvidence:RWDisgrowinginimportancetoevidencegenerationfornegotiationswithpayersandHealthTechnologyAssessment(HTA)bodies. AnIQVIAanalysisshowedRWDuseinHTAreportsubmissionsincreasedfrom6%in2011to39%in20214.Thisroleisparticularlysignificantfortargetedtherapies,wheremarketadoptionistypicallyslower,makingprospectiveobservationalstudiesalessthanidealsolutionforpost-launchevidencegeneration. Drivingmarketpenetrationbypromotingbiomarkertestingpractices:RWEisinstrumentalinunderstandingandpromotingbiomarkertestingpractices,whicharecrucialforintroducingtargetedtherapiestothemarket.Wehavewitnessedlargevariationsinadoptionratesbetweencancertargetedtherapies,mainlyduetodifferencesintestingpractices.RWDstudiesarevitalforengagingKeyOpinionLeaders(KOLs)andincreasingproductawarenesswithinthemedicalcommunity. Challenge:RWEwithnovelbiomarkers UtilizingRWDfortargetedtherapiesisanimperativeforinnovativepharmacompanies,yetitisataskriddledwithchallenges.AccordingtoanIQVIAOncologyDynamicsreport,cancerpatientsintheEU5countriesaretestedforanaverageofonebiomarker,predominantlyinspecificindicationssuchaslungandbreastcancer5.Despitetherapidly growinglistofrelevantbiomarkers,therateofclinicaladoptionisconsiderablyslower,makingitdifficult forcompaniestogenerateRWEfortheirtargeteddrugsrelyingonbiomarkersnotcommonlytestedinroutineclini