全球视野下：ADC的trial策略

ADCClinicalTrialStrategy ShuhuaZhou（周淑华）PhDCiteline Mar.9th2024 Outline 1.ADC领域的市场空间和临床试验启动趋势 2.临床可行性评估（患者数量，临床时长，Patientjourney） 3.ADC临床开发的适应症和患者、主要终点选择 4.ADC临床试验的Combo趋势 ADCDrugswithHighLikelihoodofApproval Source:Biomedtracker®andPharmapremia® ADCDrugsWWsalesforecast 35,000.0 WWSalesbyIndication($m)CAGR26.9% 1.00% IndicationMarketShareChange/2028(m$) 30,000.0 31,227.5 0.80% Breastcancer 25,000.0 IndicationMarketShare 0.60% Indicationsales(m$) 20,000.0 15,000.0 10,000.0 0.40% 0.20% Bladdercancer Non-smallcelllungcancer(NSCLC) Non-Hodgkinlymphoma(NHL) 26,516.6 21,934.3 17,501.4 13,585.1 10,118.3 7,469.8 Hodgkinlymphoma Ovariancancer 5,000.0 0.00% -2000-1000010002000300040005000600070008000 0.0 2022202320242025202620272028 -0.20% SalesChangeOverTime Source:EvaluatePharma[Accessed2024] ADCDrugsTrialsbyStartDate 250 IV III/IV IIIII/IIIIII/II I 200 TrialNumber 150 100 50 0 1995 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022 2023 Source:Trialtrove Outline 1.ADC领域的市场空间和临床试验启动趋势 2.临床可行性评估（Patientjourney，患者数量，临床时长与成本） 3.ADC临床开发的适应症和患者、主要终点选择 4.ADC临床试验的Combo趋势 PatientDemographicsandPatientJourney •Betterunderstandingofpatientdynamicstoassistwithpharmacompanystrategicplanning–improveclinicaltrialdesignforpipelinedrugsandpatienttargetingforlauncheddrugs •Reimbursementclaimsdataanalysiscanalsobeusedtoinformpharmacompaniesaboutthepatientjourney,e.g.,whetherpatientsmostlyseekcareathospitalsorclinics,referralpatterns,treatingphysicianspecialties,etc. •Suchanalysisisvitalwhenplanningclinicaltrialsandassessingthepotentialfuturevalueofaproduct,particularlywhenothersourcesofepidemiologydataareunavailableorunreliable,asisoftenthecasewithrarediseases •Reimbursementclaimsdataanalysisenablestheinvestigationofpatientdemographicsformoreaccurateestimationofthepatientpopulationwithaparticulardisease,aswellaspatientdistributionbystateorothergeographicregion Description Examples TurnRealWorldData(RWD)intoInsights Multipledatasetsenterourplatform Dataiscleansedandharmonized Norstelladataassetsareappended Epidemiologydata Combineddatapowersproductsandservices 300M+ Longitudinalpatients 2.1M+ HCPs Matching&normalizationintegrationprocess AdditionalDatasets Formulatory&enrollmentdataHEORdata BetterInsightsFasterRelatedto:ClinicalTrialPlanningProtocolOptimization Site&PISelection 32B+Claims200M+AnnualPatients PayerDetailAvailable SpecificsbybenefitplanRemittanceswithallowedamounts SubmissionswithPriorDenialswithrationalespecificityAuthorization(PA),QuantityLimitDenialreasons (QL),andStepTherapy(ST)Rebates,coupons,deductibles,co-specificityinsuranceandfullpayment amounts 98%ofPayers AffiliationHierarchy Differentiationoftreatmentsettings-(inpatient/outpatient) Line-itemchargedetails Behaviors&decisionsofphysicians,assistants,nursepractitioners,registerednurses,andotherhealthcarestaff Granularity +7KHospitals +5,600HealthSystems +1,500ACOs +6.5MAffiliations Granularity NPI,First/LastName IDNAffiliations +2.1MHCPs PatientVolumesGranularity 300M+totalpatientsfrom2017PatientGeographyatZiplevelforwardSite-of-careaddress 200M+patientsannuallyDemographics(age,race,gender) Out-of-pocketcosts CoveragebyPayerTypeWeeklydatarefreshes ~80%ofcommerciallives ~60%ofMedicarelives ~40%ofMedicaidlives +300MPatients 300M+OverallPatients(2017–Present) Labs Rx+ EMR Sites&investigatorsdata ClinicaltrialsdataPublicationsdata CommercialStrategy BenchmarktoADCPhaseI&PhaseIIITrialsinLast10Years PhaseI 25 NumberofTrials 20 15 10 5 0 EnrollmentDuration(Actualsonly) EnrollmentDuration(months) Accrual(Actualsonly) 50 NumberofTrials 40 30 20 10 0 Accrual ReportedSites 80 70 NumberofTrials 60 50 40 30 20 10 0 ReportedSites Mean: 27.4 Median: 26.45 Min: 1.84 Max: 72.65 Mean: 58.05 Median: 36.5 Min: 3 Max: 292 Mean: 12.3 Median: 7 Min: 1 Max: 96 PhaseIII EnrollmentDuration(Actualsonly) 1012 8 910 NumberofTrials NumberofTrials 78 6 56 4 34 22 1 00 EnrollmentDuration(months) Accrual(Actualsonly) Accrual ReportedSites 45 40 35 NumberofTrials 30 25 20 15 10 5 0 ReportedSites Mean: 26.68 Median: 23.82 Min: 11.83 Max: 67.16 Mean: 546.47 Median: 498 Min: 106 Max: 1,500 Mean: 152.38 Median: 111 Min: 1 Max: 1,145 Trialtrove®,Mar2024 ADCTrialEnrollmentEfficiencyinTop10CountryLast10Years Country NumberofTrials AverageActual AverageActual PhaseIPhaseIII Country NumberofTrials Pts/Site/Mo Pts/Site/Mo UnitedStates 146 0.68 China 71 0.14 China 103 0.61 UnitedStates 69 0.13 Spain 46 0.19 Italy 60 0.13 Japan 39 1.28 Spain 59 0.14 France 35 1.62 Germany 58 0.13 Australia 34 0.15 Canada 56 0.15 SouthKorea 29 0.17 UnitedKingdom 55 0.13 Canada 27 0.20 France 54 0.13 Italy 19 0.31 SouthKorea 53 0.16 UnitedKingdom 18 0.32 Australia 49 0.13 Trialtrove®,Mar2024 TrialCostMeanBenchmark Indication TotalPIIITrialCostMeanBenchmark($m) TotalPIITrialCostMeanBenchmark($m) TotalPITrialCostMeanBenchmark($m) Non-smallcelllungcancer