从那他珠单抗上市、撤市看美国和英国新药风险管理经验  

Risk Management Experience for Innovative Drugs of FDA and MHRA Derived from the Marketing or Withdrawal of Natalizumab

作　　者：李幼平[1] 李媛媛[1] 王莉[1] 

机构地区：[1]四川大学华西医院循证医学与临床流行病学教研室,成都610041

出　　处：《中国循证医学杂志》2010年第7期811-816,共6页Chinese Journal of Evidence-based Medicine

摘　　要：目的以那他珠单抗为例探讨美国和英国对高风险新药的管理办法和实施效果,为我国创新药物及高风险上市药物的风险监测和管理提供借鉴。方法检索美国食品药品监督管理局(FDA)及英国药物与保健产吕监督署(MHRA)官方网站。纳入那他珠单抗上市、撤市及其风险管理以及药物上市、撤市、审评及审批途径等信息。结果①那他珠单抗于2004年11月在尚未结束临床试验时经FDA快速通道审批上市,2005年因发生3例进行性多灶性白质脑病(PML)撤市;后于2006年6月在"风险最小化计划"下重新上市。②该药2006年4月于严密监测下在欧洲上市,英国至今未见PML报道。③为了加速疑难危重病创新治疗药物的研发,FDA采取了3项特殊政策支持:快速通道、优先审查和加速审批。2002～2009年经快速通道审批上市的化学药及生物制剂分别达45%和74%。④英国MHRA药物监管内容包括:黄卡方案、黑三角标识、药物缺陷报告中心和药物分析资料下载。其自报及监测系统较为完善,因而能在现有条件下完成对高风险药物的严密监测,实现风险最小化。结论①FDA对高风险药物的风险最小化计划行之有效,对不可替代的创新药物研发、上市起到了政策保障作用。②MHRA将高风险上市药物的风险监测与管理整合到行之有效的上市后药物监测计划中并实行分级管理,也取得了风险最小化效果。③我国创新药物和高风险上市药物的再评价应借鉴国际化学药物尤其是生物制剂再评价和政策制定及管理运行经验,结合具体药物特点,循证制定各种评价指标和标准,不断建立和完善创新药物的风险管理体系和机制,为创新药物和高风险药物的安全使用提供政策和管理保障。Objective To investigate the regulations and implementation effect for high-risk drugs of the FDA and MHRA on the basis of natalizumab,and to provide references for the risk regulation of the innovative drugs and high-risk drugs of China.Methods We searched MEDLINE,EMBASE.com,the official website of Food and Drug Administration（FDA） and the Medicines and Healthcare Products Regulatory Agency（MHRA） for the marketing/withdrawal and risk regulation information of natalizumab as well as the relevant information of drug marketing/withdrawal and approval track.Results（1） Natalizumab was initially approved by the FDA through accelerated approval in November,2004 with the phase three clinical trial still being conducted.But in February 2005,it was withdrawn a er 3 patients developed PML.FDA resumed its marketing under a specially restricted distribution program called the Touch Prescribing Program in March,2006.（2） Natalizumab was marketed in the European Union in April 2006.No cases of PML have been reported in the UK for this drug.（3） To speed the development of drugs that treat serious diseases,the FDA developed 3 distinct and successful approaches to make such drugs available as rapidly as possible：Priority Review,Accelerated Approval,and Fast Track.The proportion of new molecular entity and new biologic approved by fast tract were 45% and 74%,respectively between 2002 and 2009.（4） Drug regulation of MHRA contained：Yellow Card Scheme,Black Triangle products,Download Drug Analysis Prints（DAPs）,and Defective Medicines Report Centre（DMRC）.Self-reporting and monitoring system were fairly perfect,thus they can fulfill the close monitoring for high-risk drugs under the existing conditions and then realize the risk-minimization.Conclusion（1） The risk-minimization program of FDA for high-risk drugs is effective,and plays a role in the policy support for the researching and marketing of irreplaceable innovative drugs.（2） The risk regulation for high-risk drugs of MHRA is integrated

关 键 词：那他珠单抗 上市 撤市 风险管理 

分 类 号：F416.72[经济管理—产业经济]