开展超适应证用药临床研究面临的问题探讨  

Discussion on problems in conducting the clinical trials on off-label use of drug

作　　者：黄晓铨 陈世耀[1,2] HUANG Xiao-quan;CHEN Shi-yao(Department of Internal Medicine, Zhongshan Hospital Affiliated to Fudan University, Shanghai 200032, China;Center of Evidence-based Medicine, Fudan University, Shanghai 200032, China)

机构地区：[1]复旦大学附属中山医院内科教研室,上海200032 [2]复旦大学循证医学中心,上海200032

出　　处：《中国药物应用与监测》2017年第1期60-62,共3页Chinese Journal of Drug Application and Monitoring

基　　金：国家科技支撑计划项目(2013BAI09B14)

摘　　要：超适应证用药在临床实践中较为普遍,全球对超适应证用药的关注度逐年增长,但开展超适应证用药的临床研究存在诸多问题。笔者针对超适应证用药如何开展临床研究和开展临床研究中存在的问题进行了深入的探讨。提出了适合开展超适应证用药临床研究的情况,超适应证临床研究申报者的争议,并解释了开展超适应证用药研究是否需要国家食品药品监督管理总局的批件,如何获得伦理委员会批件,如何进行研究的注册,获得资金支持的途径,如何做好知情同意和患者选择,以及如何选择超适应证用药的研究设计等问题。以期更好地规范临床实践中超适应证用药,开展超适应证临床研究,以减少不良事件的发生。Off-label prescriptions are common in clinical practice,and its relative researches increase globally over time.However,a lot of problems remain in conducting the clinical trials on off-label drug use.The aim of this article was to discuss how to conduct clinicaltrials on off-label use of drugs and its remaining problems.The problems were discussed as follows:when to conduct the off-label drugclinical trial;the controversy about who should be the investigators;whether the CFDA(China Food and Drug Administration)license isrequired for conducting such trials;how to obtain the permission from Clinical Ethics Committee;how to register the clinical trials on offlabeluse of drug;how to gain the funding supports;how to have your patients'consent;how to select a suitable patient population;andhow to conduct study design of clinical trial on off-label use of drug.Detailed interpretations were made into these questions.We hope tostandardize the off-label medication,conduct the proper clinical trial on off-label drug use,and reduce the occurrence of adverse events

关 键 词：超适应证 临床研究 分析 

分 类 号：R95[医药卫生—药学]