EBE-EFPIA Initiative Escher-ATMP Project Results .Escher-ATMP Project EBE-EFPIA Initiative:...

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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)regulatoryandqualityexpertiseandbusinessgoalsmostlyarentabletocatchup,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.AnkeHvelsAssistantProfessor

Prof.Dr.MariekedeBruinRegulatoryScience,UnivofCopenhagenAssistantprofessor,UtrechtUniversity

ATMPworkinggroup

BarbaraFreischemExecutiveDirector

EstherChoiVCworkinggroup,BMS