Challenges with advanced therapy medicinal products and how to meet them | Nature Reviews Drug Discovery

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ChallengeswithadvancedtherapymedicinalproductsandhowtomeetthemTheCommitteeforAdvancedTherapies(CAT)
Abstract

Advancedtherapymedicinalproducts(ATMPs),whichincludegenetherapymedicinalproducts,somaticcelltherapymedicinalproductsandtissue-engineeredproducts,areatthecuttingedgeofinnovationandofferamajorhopeforvariousdiseasesforwhichtherearelimitedornotherapeuticoptions.Theyhavethereforebeensubjecttoconsiderableinterestanddebate.FollowingtheEuropeanregulationonATMPs,aconsolidatedregulatoryframeworkfortheseinnovativemedicineshasrecentlybeenestablished.CentraltothisframeworkistheCommitteeforAdvancedTherapies(CAT)attheEuropeanMedicinesAgency(EMA),comprisingamultidisciplinaryscientificexpertcommittee,representingallEUmemberstatesandEuropeanFreeTradeAssociationcountries,aswellaspatientandmedicalassociations.Inthisarticle,theCATdiscussessomeofthetypicalissuesraisedbydevelopersofATMPs,andhighlightstheopportunitiesforsuchcompaniesandresearchgroupstoapproachtheEMAandtheCATasaregulatoryadvisorduringdevelopment.

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Figure1:ExpertiseinthefieldofadvancedtherapymedicinalproductsdeclaredbymembersandalternatesoftheCommitteeforAdvancedTherapies.

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Acknowledgements

TheCATChairmanwishestothankthoseCATcolleagueswhotookas"Rapporteurs"theleadinthediscussionsofthesubsectionsofthisarticletocoordinatetheparticipationoftheentireCommittee,namely:P.Salmikangas(CATVice-ChairandFinnishCATmember)andA.Tsiftsoglou(GreekCATmember)forCBMPs;T.VandenDriessche(AlternateforClinicians"Representative)forthegenetherapysection;W.Cheng(SwedishCATAlternate)forthesectiononcombinedATMPs;andP.Celis(EMACATScientificSecretariat)forthesectiononhowtheCATisinvolvedinthedevelopmentofATMPs.

AuthorinformationAffiliationsC.K.S.isChairmanoftheCAT;HeadofDivisionEUCooperation/MicrobiologyatthePaul-Ehrlich-InstitutFederalAgencyforSeraandVaccines,Paul-Ehrlich-Institut,Paul-Ehrlich-Str.51-59,D-63225Langen,Germany;andattheTwincoreCenterforExperimentalandClinicalInfectionResearch,Feodor-Lynen-Str.7,30625Hannover,Germany.聽schci@pei.deChristianK.SchneiderHeadofDivisionEUCooperation/MicrobiologyatthePaul-Ehrlich-InstitutFederalAgencyforSeraandVaccines,Paul-Ehrlich-Institut,Paul-Ehrlich-Str.51-59,D-63225Langen,GermanyChristianK.SchneiderP.C.istheScientificSecretaryoftheCATandtheScientificAdministrateroftheEuropeanMedicinesAgency,7WestferryCircus,CanaryWharf,LondonE144HB,UK.patrick.celis@emea.europa.euPatrickCelisConsortiaTheCommitteeforAdvancedTherapies(CAT)ChristianK.Schneider,聽PaulaSalmikangas,聽BerndJilma,聽BrunoFlamion,聽LyubinaRachevaTodorova,聽AnnaPaphitou,聽IvanaHaunerova,聽ToivoMaimets,聽Jean-HuguesTrouvin,聽EgbertFlory,聽AsteriosTsiftsoglou,聽Bal谩zsSarkadi,聽KolbeinnGudmundsson,聽MauraO"Donovan,聽GiovanniMigliaccio,聽J膩nisAnc膩ns,聽RomaldasMa膷iulaitis,聽Jean-LouisRobert,聽AnthonySamuel,聽JohannesH.Ovelg枚nne,聽MaritHystad,聽AndrzejMariuszFal,聽BeatrizSilvaLima,聽AncaStelaMoraru,聽PeterTur膷谩ni,聽RobertZorec,聽SolRuiz,聽Lennart脜kerblom,聽GopalanNarayanan,聽AlastairKent,聽FabriziaBignami,聽J.GeorgeDickson,聽DietgerNiederwieser,聽Mar铆a-AngelesFiguerola-Santos,聽IlonaG.Reischl,聽ClaireBeuneu,聽RosenGeorgiev,聽MariaVassiliou,聽AlenaPychova,聽MetteClausen,聽TainaMethuen,聽SophieLucas,聽MartinaSch眉ssler-Lenz,聽VasiliosKokkas,聽ZsuzsannaBuz谩s,聽NiallMacAleenan,聽MariaCristinaGalli,聽AijaLin膿,聽JolantaGulbinovic,聽GuyBerchem,聽MariuszFr膮czek,聽MargaridaMenezes-Ferreira,聽NelaVilceanu,聽Mikul谩拧Hrubi拧ko,聽PetraMarinko,聽MarcosTim贸n,聽WingCheng,聽GeorgeAndrewCrosbie,聽NickMeade,聽MichelinoLipuccidiPaola,聽ThierryVandenDriessche,聽PerLjungman,聽LuciaD"Apote,聽OlgaOliver-Diaz,聽IsabelB眉ttel聽聽PatrickCelisEthicsdeclarationsCompetinginterests

FordetailsofcompetingfinancialinterestsforeachmemberpleaseseetheCommitteeforAdvancedTherapeuticswebsite(http://www.ema.europa.eu/htms/general/contacts/CAT/CAT_members.html).

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EMEAInnovationTaskForce

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EMEAScientificGuidelinesforBiologicals,includingguidelinesspecifictoATMPs:

EMEAMultidisciplinaryGuidelines,includingguidelinesspecifictoATMPs

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Schneider,C.,Salmikangas,P.,Jilma,B.etal.Challengeswithadvancedtherapymedicinalproductsandhowtomeetthem.NatRevDrugDiscov9,195鈥?01(2010).https://doi.org/10.1038/nrd3052

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IssueDate:March2010

DOI:https://doi.org/10.1038/nrd3052

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