真实世界数据作为外部对照用于药械临床评价的特殊考虑  

The special consideration for using the real-world data as external control in clinical evaluation

作　　者：王雨宁 姚明宏 任燕[1,2,3] 徐嘉悦 贾玉龙 林凯 李耀华 魏春敏 孙鑫 Wang Yuning;Yao Minghong;Ren Yan;Xu Jiayue;Jia Yulong;Lin Kai;Li Yaohua;Wei Chunmin;Sun Xin(Chinese Evidence-based Medicine Center,West China Hospital,Sichuan University,Chengdu 610041,China;National Medical Products Administration Key Laboratory for Real World Data Research and Evaluation in Hainan,Chengdu 610041,China;Sichuan Center of Technology Innovation for Real World Data,Chengdu 610041,China;Hainan Medical Products Administration,Haikou 570216,China;Center for Adverse Drug Reactions Monitoring of Boao Pilot Zone of Hainan Province,Haikou 570216,China)

机构地区：[1]四川大学华西医院中国循证医学中心,成都610041 [2]国家药品监督管理局海南真实世界数据研究与评价重点实验室,成都610041 [3]四川省真实世界数据技术创新中心,成都610041 [4]海南省药品监督管理局,海口570216 [5]海南省博鳌先行区药品不良反应监测中心,海口570216

出　　处：《中华流行病学杂志》2023年第2期335-340,共6页Chinese Journal of Epidemiology

基　　金：四川省青年科技创新研究团队(2020JDTD0015);四川大学华西医院学科卓越发展1·3·5工程项目(ZYYC08003);中央级公益性科研院所基本科研业务费专项(2020YJSZX-3);美国中华医学基金会项目(CMB19-324);四川省中央引导地方科技发展专项(2022ZYD0127);国家自然科学基金青年科学基金(72204173)。

摘　　要：随机对照试验(RCT)被视为药械临床疗效评价的“金标准”。但针对罕见、重大且无有效治疗方式等疾病,考虑伦理、成本等因素,RCT并不适宜。此时,采用真实世界数据(RWD)作为试验的外部对照支持药械临床评价,可降低患者招募难度、缩减研发时间及成本。本文基于国内外最新发布的RWD有关的指导原则,并结合团队前期研究经验,介绍了采用RWD作为外部对照用于支持药械临床评价的常见应用场景、数据来源、研究设计、外部对照选择基本原则和统计分析方法,以期为相关学者、申办方开展RWD研究提供参考与借鉴。Randomized controlled trials(RCT)have long been considered the gold standard for assessing clinical efficacy.However,RCT are inappropriate for some diseases due to related ethical issues and costs,such as rare diseases that are seriously life-threatening but without adequate treatment.Using real world data(RWD)as external control for RCT could make recruitment less complicated and reduce time and cost.This paper introduces common application scenarios,data sources,study design,basic principles,and statistical methods of RWD as an external control based on the latest guidelines related to RWD and combined with our team's previous research experience.This study could provide references for scholars and sponsors who want to conduct RWD research.

关 键 词：临床试验 真实世界数据 外部对照 真实世界证据 

分 类 号：R95[医药卫生—药学]