真实世界证据与随机对照试验:RCT DUPLICATE项目启动、实施、进展解读与启示(一)  

Real-world evidence and randomized controlled trials:the initiation,implementation,progress interpretation and revelation of RCT DUPLICATE(part 1)

作　　者：石舒原 刘佐相 赵厚宇 聂晓璐[1,3] 符祝 宋海波 姚晨 詹思延[1,8] 孙凤[1,4] Shi Shuyuan;Liu Zuoxiang;Zhao Houyu;Nie Xiaolu;Fu Zhu;Song Haibo;Yao Chen;Zhan Siyan;Sun Feng(Department of Epidemiology and Biostatistics,Peking University School of Public Health,Beijing 100191,China;China Institute of Rehabilitation Sciences,Center for Prevention and Control of Disability of China Disabled Persons Federation,Beijing 100068,China;Center for Clinical Epidemiology and Evidence-based Medicine,Beijing Children's Hospital,Capital Medical University,National Center for Children's Health,Beijing 100045,China;Hainan Institute of Real World Data,the Admonistration of Boao Lecheng International Medical Tourism Pilot Zone,Lecheng 571437,China;Center for Drug Reevaluation,National Medical Products Administration,Beijing 100022,China;Key Laboratory for Research and Evaluation of Pharmacovigilance,National Medical Products Administration,Beijing 100022,China;Peking University Clinical Research Institute,Beijing 100191,China;Clinical Epidemiology Research Center,Peking University Third Hospital,Beijing 100191,China)

机构地区：[1]北京大学公共卫生学院流行病与卫生统计学系,北京100191 [2]中国康复科学所,中国残联残疾预防与控制中心,北京100068 [3]国家儿童医学中心,首都医科大学附属北京儿童医院临床流行病与循证医学中心,北京100045 [4]海南省真实世界数据研究院,博鳌乐城国际医疗旅游先行区管理局,乐城571437 [5]国家药品监督管理局药品评价中心,北京100022 [6]国家药品监督管理局药物警戒研究与评价重点实验室,北京100022 [7]北京大学临床研究所,北京100191 [8]北京大学第三医院临床流行病学研究中心,北京100191

出　　处：《中华流行病学杂志》2022年第11期1828-1834,共7页Chinese Journal of Epidemiology

基　　金：国家自然科学基金(72074011);中国药品监管科学行动计划第二批重点项目([2021]37-10);海南省博鳌乐城先行区真实世界研究专项计划(HNLC2022RWS012)。

摘　　要：近些年,医疗产品监管机构开始重新审视真实世界证据(RWE)对监管决策的潜在价值。RWE能否代替金标准随机对照试验(RCT)产生的证据尚不确定。哈佛大学研究团队于2018年发起了RCT DUPLICATE项目,旨在利用医疗索赔数据库模拟30个RCT,以探索效力-效果差距的量化方法并解释其潜在来源,增强RWE的可信度。本文回顾了RCT DUPLICATE项目的产生背景,重点介绍RCT DUPLICATE项目的研究目的、研究设计和实施流程,以期帮助国内学者更好地理解RWE的适用范围和应用价值。In recent years,researchers,pharmaceutical companies,and political makers gradually using more real-world data(RWD)to produce real-world evidence(RWE)for policy-making.A research team of Harvard University launched the RCT DUPLICATE project in 2018,aiming to replicate 30 randomized controlled trials using the medical claims database in order to explore methods for quantifying the efficacy-effectiveness gap and explain its potential sources,to enhance the credibility of the RWE.This paper reviews the background of RCT DUPLICATE Initiative,highlights the research purposes,research design and implementation process of the RCT DUPLICATE Initiative,to help domestic scholars better understand the scope and application value of RWE.

关 键 词：真实世界证据 真实世界数据 模拟 

分 类 号：R195[医药卫生—卫生统计学]