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PERSPECTIVEARTICLEpublished:14February2012doi:10.3389/fphar.2012.00012AdvancedtherapymedicinalproductsandexemptionstotheRegulation1394/2007:howconfidentcanwebe?AnexploratoryanalysisPhilippeVanWilder*TiGenixNV,Leuven,BelgiumEditedby:IñakiGutiérrez-Ibarluzea,BasqueOfficeforHealthTechnologyAssessment,SpainReviewedby:DominiqueJ.Dubois,UniversitéLibredeBruxelles,BelgiumSuyashPrasad,BioMarinPharmaceutical,USA*Correspondence:PhilippeVanWilder,TiGenixNV,ResearchparkHaasrode1724,Romeinsestraat12,bus2,3001Leuven,Belgium.e-mail:philippe.vanwilder@tigenix.comThemarketauthorizationprocedureformedicinalproductsforhumanuseisrelyingontheirdemonstratedefficacy,safety,andpharmaceuticalquality.Thisappliestoallmed-icinalproductswhetherofchemicalorbiologicalorigin.SinceOctober2009,thefirstadvancedtherapymedicinalproduct(ATMP)hasbeenauthorizedthroughthecentralizedprocedure.ATMPsaregenetherapymedicinalproducts,somaticcelltherapymedicinalproductsortissue-engineeredproducts.AnappropriateATMP–RegulationisdealingwithATMPrequirements.TwoexemptionsareforeseentotheATMPRegulation:(a)Products,whichwerelegallyontheCommunitymarketwhentheRegulationbecameapplicable,shouldcomplytotheRegulationbyDecember30,2012.(b)Thehospitalexemptionrulefornon-routineproductsforanindividualpatient.InthisworkweexploredwhethertheactualapplicationoftheRegulationonATMPsisinlinewiththeaimoftheRegulationintermsofguaranteeingthehighestlevelofhealthprotectionforpatients.BasedontheanalysisoftherelativeefficacyoftheonlyECauthorizedATMPanditsexemptedalternatives,thereisevidenceagainstthisRegulation1394/2007assumption.Keywords:advancedtherapy,Regulation1394/2007,exemption,patientoutcomeINTRODUCTIONThemarketauthorization(MA)procedureformedicinalprod-uctsforhumanuseisrelyingontheirdemonstratedefficacy,safety,andpharmaceuticalquality(TheEuropeanParliamentandtheCounciloftheEuropeanUnion,2001).Thisappliestoallmedicinalproductswhetherofchemical(e.g.,bloodpres-sureloweringdiuretic)orbiological(e.g.,anti-inflammatorymonoclonalantibody)origin.Modernbiotechnologymedici-nalproductsobtainmarketapprovalthroughthecentralizedprocedureasdetailedintheECRegulation726/2004(TheEuropeanParliamentandtheCounciloftheEuropeanUnion,2004).Since2008,a“lexspecialis”–Regulation(EC)No1394/2007(TheEuropeanParliamentandtheCounciloftheEuropeanUnion,2007)–appliestoadvancedtherapymedicinalprod-ucts(ATMPs);theseATMPsarepharmaceuticalswithhighcom-plexity(TheCommitteeforAdvancedTherapies(CAT)andtheCATScientificSecretariat,2010)linkedtotheirdevelopment,manufacturing,oradministrationprocess.TheRegulationhighlightsthefollowing:•ItprovidesanexplicitATMPdefinition:ATMPsaregenetherapy,somaticcelltherapy,ortissue-engineeredmedicinalproducts.•AnATMPmustcomplywiththeexistingMArequirements(quality,safety,andefficacy)andthepost-marketingpharmaco-vigilancerules.ForMA,thecentralizedprocedureismandatory:itaimstopoolCommunityexpertiseandensureahighlevelofscientificevaluationandfacilitateaccesstomarket.•BecauseofthecomplexityofATMPs,anewCommitteeforAdvancedTherapies(CAT)hasbeeninstalled.TheCAT’smainresponsibilitiesare:•ThemandatoryevaluationofMAapplicationsbyprovid-ingopinionstotheCommitteeforMedicinalProductsforHumanUse(CHMP);theCHMPmayadoptorrefusetheCATopinion.•Theoptionalscientificcertification(art.18)ofqualityandnon-clinicaldataofaproposedATMP-compoundindevelopment.•TheoptionalscientificrecommendationonATMP-classification(art.17),priortotheirclinicaldevelopment.TheCAT(TheCommitteeforAdvancedTherapies(CAT)andtheCATScientificSecretariat,2010)isamultidisciplinaryscientificexpertcommittee:italsofocusesonthescientificdevelopmentsinthefield.Thereisnodoubtaboutthehugescientific,regula-tory,andethicalchallengestriggeredbythesecomplexproductsandaspecificexpertcommitteeforATMPsisnecessarytodealwiththesechallenges(similartothecreationoftheCommitteeonOrphanMedicinalProductsfordrugsusedinrarediseases)andbeneficialtoallrelevantpublicandprivatestakeholders.•TheTissuesandCellsDirective(2004/23/EC)appliestodona-tion,procurementandtestingofhumantissuesandcells.•TheRegulationdefinesthepre-andpost-authorizationrequire-ments:GMPandGCPstandards,productfollow-uponefficacyandsafety,riskmanagementplan,andtraceability.|Volume3|Article12|1VanWilderATMP-exemptionsandpatienthealthprotection•TheRegulationalsoprovidesincentivesforapplicantsbyoffer-ingscientificadviceatvariousdevelopmentstepsatsub-stantiallyreducedfees,mainlytosmall-andmedium-sizedenterprisesandhospitals.TheaimofthisATMP–Regulationistoofferaconsolidatedregulatoryframeworkfortheseinnovativemedicinesanditwasdesigned(EuropeanMedicinesAgency,2011a)to“...ensurethefreemovementofthesemedicineswithintheEuropeanUnion(EU),tofacilitatetheiraccesstotheEUmarket,andtofosterthecompetitivenessofEuropeanphar-maceuticalcompaniesinthefield,whileguaranteeingthehighestlevelofhealthprotectionforpatients.”TwoexemptionsareforeseentotheATMPRegulation:1.Products,whichwerelegallyontheCommunitymarketonDecember30,2008(whentheRegulationbecameapplicable),shouldbecomplianttotheRegulationrequirementsnolaterthanDe
本文标题:欧盟ATMP分析
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