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EBE-EFPIA Initiative
Escher-ATMP Project ResultsRenske ten Ham, MSc
Division of Pharmacoepidemiology and Clinical Pharmacology - Utrecht University
Escher-ATMP Project
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
OverallAim:ExaminefactorsassociatedwithsuccessfulATMPdevelopmentandcommercialisationin Europe.
Workpackage1
Survey:Provideoverviewofproductanddevelopercharacteristicsandidentifyexperiencedchallenges.
Workpackage2
Interviews:SubstantiatetheobservedfindingsfromWP1byidentifyingchallengeroot-causes.
ATMP Landscape in Europe
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
271 DevelopersIdentified38% SurveyResponse
ATMPType
46% 53%
1%
40%
59%
1%
GeneTherapy CellbasedTherapy
CombATMP
Fullcohort
Respondents
In201665%of
developersareSMEs,35%arelargedevelopers.
ATMP Landscape in Europe
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
271 DevelopersIdentified38% SurveyResponse
DeveloperDevelopmentStage
In201665%of
developersareSMEs,35%arelargedevelopers.
41
106 7
EarlyClinicalStage(phase
I-II)
LateClinicalStage(phase
III)
RegulatoryApprovalAppl
ProductCom
Survey Results
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
ExperiencedChallenges– Grouped
0%
25%
50%
75%
100% Otherchallenge(s)
HRMchallenge(s)
Clinicalchallenge(s)
Financialchallenge(s)
Scientificchallenge(s)
Technicalchallenge(s)
Regulatorychallenge(s)
Top5ExperiencedChallenges
MeetingCountrySpecificRequirements
ProductManufacturing
ClinicalTrialDesign
MAADossierContentUncertainty
QualityStandards
16%
6%
15%
8%
5%
1
2
3
4
5MAA=MarketAuthorisationApplication
Survey Results - Quotes
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
“IMPDneededresearchlevel(…)becauselegislationwasnewandnobodyhadacluehowto
doanimaltesting”
“Conductingregulatoryroadshowtomeetwithregulatorybodiesto
understandrequirementsandobtainalignment”
“LackofGMPgraderawmaterials”
“SlowCTAandGMOassessment”
“Findingappropriateclinicalinvestigationsites”
“Forregulatoryacceptancereasonstheearlystageclinicaltargetgroupwithinthepatientpopulationdiffersfromthefinalpatienttargetgroup”
Combining Survey Results with Interviews
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
ManydifferentchallengingandfacilitatingfactorsidentifiedincommercialEUATMPdevelopment.Twoelementscausingmanyhurdlesare:
ThedisconnectbetweenEuropean(highlevel)regulationandnationalhurdles.
CapabilitiesandbusinessmodelatthestarthaveeffectthroughoutATMPdevelopmentsuccess.
Disconnect between European (high level) regulation and national hurdles
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
CommercialdevelopersfindEuropeaneffortssofarhelpedATMPdevelopmentmoveforward.However,localinterpretationandexecutionleadstomanyadditional(duplicate)informationrequests,delaysandincreasedcost.
Smallcompaniesdonothavetheresourcestoaddresscountryspecificrequirements.Also,highdatarequestburdendrivespreferenceofglobaldeveloperstolaunchfirstinUSorAsia.
NationalAuthoritieshavedifferentinterpretationsofrequirementsforClinicalTrialApplications,GeneticModifiedOrganismlegislation,HospitalExemptionandGoodManufacturingPractice.Inexperienceandun-alignmentaremostmentioned.
Capabilities and business model at the start have effect throughout ATMP development success.
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
ATMPdevelopmentismuchmorecomplexthantraditionaldrugdevelopment.Developerswhostartwithout(in-house)regulatoryandqualityexpertiseandbusinessgoalsmostlyaren’tabletocatchup,especiallyacademicspin-offandSMEs.
Arealisticbusinessmodeladdressesatleastthefollowingfactors:- Shortandlongtermdevelopmentgoals- Regulatoryandmarketaccessstrategy- Realisticreturnoninvestmentexpectationandprediction
Payers/HTAneedtojoinregulatorsininteractionstodecreasehighperceivedreimbursementcriteriaanduncertaintytomaintainpromiseofgrowingEUATMPmarket.
Concluding: Escher-ATMP project
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
Europeaneffortsareverywellreceivedbydevelopers.Compared,nationalauthoritieshavenotmaturedandneedtobridgegapsbetweenEuropeandeachother.
• MoreharmonizationofCTA,GMOandGMPrequirementsbetweenmemberstatesisneededtofurtheradvanceEUATMPdevelopmentandcompetewithotherinternationalmarkets.
ATMPdevelopmentrequiresearlierplanningandspecialisedskillscomparedtotraditionaldrugdevelopment.StartingwitharealisticbusinessmodelandearlyregulatoryandHTAinvolvement.
EUATMPlandscapeisinearlystages,shownbythemajorityofcommercialdeveloperswhoareinearlyclinical(phaseI-II)productdevelopmentalstages.
Concluding: Escher-ATMP project
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
Stakeholder specific recommendations
• LocalRegulators: NeedtobridgeknowledgegapbetweennationalandEuropeanregulatoryauthorities.Countryspecificrequirementsareamajorhurdlefordevelopers.
Payers/HTA: Needtocollaboratewithdevelopersandregulatorstoaddresslastmajorandmostlyunaddressedhurdle.ATMPHTAandreimbursementisinitsinfancy.
SMEs:EarlyonincludeGMPgradestandards,regulatoryandqualityknowledgeandbuildrealisticbusinessplan.Partnershipswithexperiencedpartytoacceleratedevelopmentandfunding.
• EuropeanRegulators:OntherighttracktoshapeEuropeanATMPlandscape,includelocalauthoritiesandpayers/HTAintheprocess.
Largecompanies:MosthadsamesteepATMPlearningcurveasSMEsbutmoreexperienceinnon-ATMPpharmaceuticaldevelopment.PartnerwithSMEstofillpipelineandaccelerateinnovation.
Acknowledgements
EBE-EFPIAInitiative:Escher-ATMPProjectResultsByRensketenHam,MSc- DivisionofPharmacoepidemiologyandClinicalPharmacology- UtrechtUniversity
Dr.AndreBroekmansExecutiveDirector
Prof.Dr.OlafKlungelEpidemiologicalMethods
Dr.JarnoHoekmanAssistantProfessor
Dr.AnkeHövelsAssistantProfessor
Prof.Dr.MariekedeBruinRegulatoryScience,UnivofCopenhagenAssistantprofessor,UtrechtUniversity
ATMPworkinggroup
BarbaraFreischemExecutiveDirector
EstherChoiVCworkinggroup,BMS
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