Abstract
周佳薇,黄丽红,尤东方,陈峰,赵杨.利用现实世界数据仿真临床试验:目标试验的发展与应用[J].Chinese journal of Epidemiology,2024,45(2):279-285
利用现实世界数据仿真临床试验:目标试验的发展与应用
The emulation of clinical trials with real-world data: development and application of target trial
Received:August 21, 2023  
DOI:10.3760/cma.j.cn112338-20230821-00081
KeyWord: 仿真目标试验  观察性研究  证据质量  临床试验
English Key Word: Target trial emulation  Observational study  Quality of evidence  Clinical trial
FundProject:国家自然科学基金(82173620,82373690,82273733,82204156);江苏省高校优势学科建设工程
Author NameAffiliationE-mail
Zhou Jiawei Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, China  
Huang Lihong Department of Biostatistics, Zhongshan Hospital, Fudan University, Shanghai 200032, China  
You Dongfang Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, China  
Chen Feng Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, China  
Zhao Yang Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, China yzhao@njmu.edu.cn 
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Abstract:
      临床试验是评价干预措施疗效和安全性的金标准,但存在花费大、耗时长等限制。现实世界数据可为比较性研究提供强大的数据基础,但研究质量参差不齐。本文介绍了仿真目标试验,其利用现实世界数据,按照临床试验的设计,事先定义暴露和结局、设立纳入排除标准、确定时间零点、估计样本量和制定统计分析计划等,以期提高观察性研究的证据等级,并初步讨论仿真目标试验的证据等级评价标准,通过案例解读仿真目标试验。
English Abstract:
      Clinical trial is the gold standard for evaluating the efficacy and safety of interventions; however, it is limited by high costs and long time. Real-world data (RWD) can provide a robust data basis for comparative research, but the quality is uneven. This review introduces the target trial emulation, in which researchers, using RWD and following the design of clinical trials, define exposure and outcome in advance, set eligibility criteria, determine the time zero, estimate sample size, and plan statistical analysis, to enhance the quality of evidence for observational studies. This review preliminarily discusses the standard of evidence quality evaluation in target trial emulation. Then, the target trial emulation is shown through case interpretation.
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