Innovation in Chemistry, Manufacturing, and Controls—A Regulatory Perspective From Industry ScienceDirect

InnovationinChemistry,Manufacturing,andControls—ARegulatoryPerspectiveFromIndustry-ScienceDirect
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AdvancedJournalofPharmaceuticalSciencesVolume108,Issue7,July2019,Pages2207-2237ReviewInnovationinChemistry,Manufacturing,andControls—ARegulatoryPerspectiveFromIndustryAuthorlinksopenoverlaypanelNinaS.Cauchon1

ShirleyOghamian1SorayaHassanpour12MichaelAbernathy1Showmore

https://doi.org/10.1016/j.xphs.2019.02.007GetrightsandcontentUnderaCreativeCommonslicenseopenaccessAbstract

Thisreviewdescribesthelandscapeofnovelmodalitiessuchascellandgenetherapies,viruses,othernovelbiologics,oligomers,andemergingtechnologies,includingmodernanalytics.Wesummarizetheregulatoryhistoryandrecentlandmarkdevelopmentsinsomemajormarketsandexaminespecificchemistry,manufacturing,andcontrols(CMC)challenges,includingsuggestionsforexplorationofpotentialscience-basedapproachesinsupportofregulatorystrategydevelopmentfromanindustryperspective.Inaddition,weevaluatetheeconomicfactorscontributingtopatientaccesstoinnovationanddiscusstheimpactofregulation.Thereisadesperateneedforaconsistentformofregulationwhereglobalapproachestoregulatorystrategiescanbeharmonized,andspecificCMCchallengescanbedealtwithusingtheappropriatescienceandrisk-basedtools.Althoughthesetoolsarewelldescribedincurrentguidancedocuments,thespecificsofapplicabilitytocomplexnovelmodalitiescanstillresultindifferingregulatoryadviceandoutcomes.Thefuturegoalsforefficientlyregulatinginnovativemodalitiesandtechnologiescouldbeaidedbymoreregulatoryharmonization,regulatoryeducation,andindustrycooperationthroughconsortia,enablingindustrytosupplykeyinformationtoregulatorsinatransparentyetwell-definedmanner,andutilizingmutuallyunderstoodrisk-benefitanalysestoproducedrugswithappropriatesafety,efficacy,andqualitycharacteristics.

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KeywordsregulatorysciencedrugstandardsindividualizeddrugtherapybiotechnologyanalysisgenetherapyimmunotherapyAbbreviationsusedAAVadeno-associatedvirusADCantibody-drugconjugateALLacutelymphoblasticleukemiaAPCantigen-presentingcellATMPadvancedtherapymedicinalproductBGTDbiologicsandgenetictherapiesdirectorateBiTEantibodyconstructbispecificT-cellengagersBLAbiologicslicenseapplicationBsIgGbispecificIgGBWPbiologicsworkingpartyCARchimericantigenreceptorCATCommitteeforAdvancedTherapiesCBERCenterforBiologicsEvaluationandResearchCDECenterforDrugEvaluationCDERCenterforDrugEvaluationandResearchCGTcellularandgenetherapyCHMPCommitteeforMedicinalProductsforHumanUseCMcontinuousmanufacturingCMCchemistry,manufacturing,andcontrolsCOCchainofcustodyCOIchainofidentityCQAcriticalqualityattributesCTLcytotoxicT-cellCTOcells,tissues,andorgansDARdrugantibodyratioDCdendriticcellDLBCLdiffuselargeB-celllymphomaEBVEpstein-BarrvirusECEuropeanCommissionEMAEuropeanMedicinesAgencyETTemergingtechnologyteamFDAUSFoodandDrugAdministrationFDCfood,drug,andcosmeticGMOgeneticallymodifiedorganismGMPgoodmanufacturingpracticeGTMPgenetherapymedicinalproductHLAhumanleukocyteantigenHLEhalf-lifeextensionHPFBhealthproductsandfoodbranchHPV-16E6humanpapillomavirustype16E6ICHInternationalCouncilforHarmonisationoftechnicalrequirementsforpharmaceuticalsforhumanuseILinterleukinIMTinnovativemanufacturingtechnologyworkinggroupINDinvestigationalnewdrugINTERACTinitialtargetedengagementforregulatoryadviceonCBERProducTsLmlisteriamonocytogenesMAMmulti-attributemethodMHLWMinistryofHealth,Labour,andWelfareNDAnewdrugapplicationNKnaturalkillerNMPAnationalmedicalproductsadministrationNOC/cnoticeofcompliancewithconditionsNSCLCnon–smallcelllungcancerOGTRofficeofthegenetechnologyregulatorPeptiCRAdpeptide-coatedconditionallyreplicatingadenovirusPDFUAprescriptiondruguserfeeactPD-L1programmeddeath-ligand1pLADDpersonalizedlive,attenuated,double-deletedPMDApharmaceuticalsandmedicaldevicesagencyPEGpolyethyleneglycolRMATregenerativemedicineadvancedtherapyRNAiRNAinterferenceR/RrelapsedorrefractoryRTRreplicationcompetentretrovirusSc-Fvsingle-chainvariablefragmentSCTMPsomaticcelltherapymedicinalproductsiRNAsmallinterferingRNAsSMEsmall-andmedium-sizedenterprisesSMRAstatemarketregulatoryadministrationSPEARspecificpeptideenhancedaffinityreceptorTALENStranscriptionactivator-likeeffectornucleasesTCRT-cellreceptorTGAtherapeuticgoodsadministrationTEPtissueengineeredproductTILtumor-infiltratinglymphocytetLLOtruncatedfragmentoflisteriolysinOTRiMtargetedmRNAmoleculeWHOWorldHealthOrganizationZFPzincfingerDNA-bindingproteinsRecommendedarticlesCitingarticles(0)

Conflictsofinterest:AllauthorsareemployeesofAmgenInc.(exceptSHwhoisaFellow).Allauthorscontributedtothewritingofthereport,andapprovedthefinalversionofthemanuscriptandthedecisiontosubmitthemanuscriptforpublication.CompleteHealthcareCommunicationsLLCprovidededitorialassistance,fundedbyAmgenInc.

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