临床试验中借用外部数据的统计学方法  

Statistical Methods for Borrowing External Data in Clinical Trials

作　　者：陈金梅 赵圆圆 朱鹏飞 陈平雁 秦国友 吴莹 CHEN Jin-mei;ZHAO Yuan-yuan;ZHU Peng-fei;CHEN Ping-yan;QIN Guo-you;WU Ying(Department of Biostatistics,School of Public Health,Fudan University;Hainan Lecheng Institute of Real World Research;BARDS,MSD China;Department of Biostatistics,School of Public Health,Southern Medical University)

机构地区：[1]复旦大学公共卫生学院生物统计学教研室 [2]海南乐城真实世界研究院 [3]默沙东研发(中国)有限公司 [4]南方医科大学公共卫生学院生物统计学系

出　　处：《中国食品药品监管》2024年第10期10-23,共14页China Food & Drug Administration Magazine

基　　金：国家自然科学基金资助项目(82273732、82473724);海南博鳌乐城国际医疗旅游先行区真实世界研究资助项目(HNLC2022RWS018);广州市基础研究计划基础与应用基础研究项目(2023A04J1106);上海市市级科技重大专项(ZD2021CY001)。

摘　　要：随着历史数据和同期外部数据等真实世界数据(RWD)资源及质量日益提升,越来越多的研究者开始关注使用外部数据为药物和医疗器械的安全性及有效性评价提供更全面有效的证据支持,特别是在难以实施大样本随机对照试验的罕见病和儿科疾病领域。因此,创新的分析方法对于高效利用这些数据变得尤为重要。在此背景下,本文旨在系统梳理并介绍临床试验中外部数据借用的研究方法,同时对比各种研究方法的优势与劣势,以期为研究者提供有价值的参考,进而推动临床试验在利用外部数据时更加高效、准确。With the ongoing improvement in the resources and quality of real-world data(RWD),including historical data and concurrent external data,an increasing number of researchers are focusing on leveraging external data to provide more comprehensive and effective evidence for evaluating the safety and efficacy of drugs and medical devices.This is particularly crucial in fields such as rare diseases and pediatrics,where conducting large-scale randomized controlled trials is often impractical.Therefore,innovative analytical methods are essential for efficiently utilizing these data.In this context,this paper aims to systematically review and introduce the research methods for borrowing external data in clinical trials,while comparing the advantages and disadvantages of various methods.The goal is to provide valuable references for researchers and promote more efficient and accurate use of external data in clinical trials.

关 键 词：真实世界研究 外部数据 药物和医疗器械 临床试验 统计分析方法 

分 类 号：R95[医药卫生—药学]