FDA建立与申请人沟通的会议制度的经验对我国新药审批的

I摘要新药的开发和审批是一个系统而复杂的科学过程。在此过程中，申办者和审评人员对新药的安全性、有效性和质量等特性和审批要求的了解程度和方式以及角度是不同的，这会导致新药研究开发进展缓慢、风险增大、审批结果不可预测及审批时间延长等诸多问题。因此，建立有效的双方沟通交流渠道是必要而且重要的。FDA从1997年实施的PDUFAII开始积极探索在新药研究开发和审批过程中建立与申办者和/或申请人沟通交流的正式会议制度，并从法律和法规层面对正式会议制度进行了规范。FDA在其指南文件中对正式会议的类型、请求、评估、会议资料的要求和提交、召开会议的程序、会议纪要的整理等均作了详细规定。申办者可以在IND提交前、I期试验结束、IIa期试验结束、II期试验结束、NDA/BLA提交前等新药开发的关键环节申请正式会议。调查数据表明，这些会议对提高新药开发的质量和上市申请的质量是非常关键的。会议制度化加快了新药的开发，有效地缩短了新药审批时间，节约了开发成本，同时也减少了无效浪费。国家食品药品监督管理局已建立了多种对申请人的咨询沟通途径，但现有的沟通渠道还存在一定的局限性和问题，还未形成对新药开发和审批的有效促进作用。在中国近期内实行SFDA(CDE)与申请人沟通会议制度化是非常必要的，而且是可行的。关键词：新药开发和审批；正式会议；FDA沟通交流会议制度；国家食品药品监督管理局；IIFDA’sExperienceinEstablishingMeetingSystemforCommunicationWithSponsorsorApplicantsandItsIlluminationonChineseNewDrugReviewandApprovalSystemAbstractDevelopmentandreviewofanewdrugisasystemic,complicated,andscience-basedprocess.Thesponsorsandreviewerscomprehendthenewdrug’scharacteristicsrelatedtoitssafety,efficacyandquality,andtechnicalrequirementsforapprovalwithdifferentapproaches,viewpointsanddegreeduringthisprocess.Thesedifferencesmayleadtomanyproblemssuchasslowprogressofnewdrugdevelopment,enlargedrisks,unpredictablereviewresultsandprolongedreviewprocess,etc.Therefore,itisnecessaryandimportanttoestablishtheefficientcommunicationchannelbetweensponsors/applicantsandreviewers.WiththeimplementationofPDUFAIIin1997,FDAexploredtoestablishtheformalmeetingsystemtocommunicatewiththesponsorsduringdevelopmentandreviewprocessofanewdrug,andregulateditwithlawandregulations.FDAprescribesthetypesofmeetings,proceduresforrequestingmeetings,contentofinformationpackages,proceduresfortheconductofmeetings,anddocumentationindetailsintheGuidance.Thesponsorscanapplyfortheformalmeetingatcriticalmilestonesindrugdevelopment,suchaspre-IND,endofphase1,endofphase2a,endofphase2,pre-BLAorNDA,etc.Surveydatashowedthatthesemeetingsarecriticalforimprovingthequalityofdevelopmentandmarketingapplication.Thesystemicmeetingspedupthenewdrugdevelopment,shortenedthereviewtime,savedthedevelopmentcost,andreducedtheuselesswaste.SFDAsetupmanychannelstocommunicatewiththeapplicants,buttherearesomelimitationandissuesatcurrentchannels.Thesecommunicationchannelsdidn’timprovethedevelopmentandreviewofnewdrugsyet.ItisnecessaryandfeasibleforSFDAactualizingmeetingsystemforcommunicationwiththeapplicantsinthenearfuture.KeyWords:DevelopmentandReviewofNewDrug;FormalMeeting;FDA;MeetingSystemforCommunication;SFDA;目录第1章序言.................................................................................................................................................1第2章FDA建立会议制度的背景、法规依据和指南文件...................................................................22.1会议制度产生的历史背景.......................................................22.2会议制度的法律和法规依据.....................................................32.3与会议制度有关的指南文件.....................................................6第3章FDA与申办者或申请人的正式会议制度介绍...........................................................................73.1会议类型.....................................................................73.2申办者或申请人提出会议请求...................................................83.3FDA对会议请求的评估........................................................103.4会议调整和取消..............................................................103.5会议资料包的内容和提交......................................................113.6预会议和与申办者或申请人的沟通..............................................133.7召开会议的程序..............................................................133.8文件整理....................................................................143.9对会议纪要的争议的解决......................................................14第4章FDA与申办者在新药开发的关键环节举行的会议.................................................................144.1IND提交前会议..............................................................154.21期结束时会议..............................................................184.32A期结束时会议..............................................................184.42期结束会议................................................................184.5NDA/BLA提交前会议..........................................................19第5章FDA实行的正式会议制度对新药审批的影响.........................................................................20第6章中国SFDA在新药审批过程中与申请人沟通的现状及影响.................................................256.1SFDA药品审评中心与注册申请人沟通的现状.....................................256.2现有的沟通渠道的局限性和存在的问题..........................................266.3现有的沟通方式和渠道对新药审评过程的影响....................................27第7章我国在药品审批过程中建立与申请人沟通交会议制度的可行性探讨..............................317.1建立与申请人沟通交流会议制度的法规依据......................................317.2建立沟通交流制度对审评资源的要求............................................32第8章对我国建立与申请人沟通的会议制度的建议........................................................................338.1沟通交流会议制度的适用范围..................................................338.2沟通交流会议的时机和内容....................................................348.3沟通交流会议的类型和流程....................................................35第9章结论.....................................................................................................