Zhou J W, Huang L L, You D F, Chen F, Zhao Y
Department of Biostatistics, School of Public Health, Nanjing Medical University, Nanjing 211166, China.
Department of Biostatistics, Zhongshan Hospital, Fudan University, Shanghai 200032, China.
Zhonghua Liu Xing Bing Xue Za Zhi. 2024 Feb 10;45(2):279-285. doi: 10.3760/cma.j.cn112338-20230821-00081.
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.
临床试验是评估干预措施疗效和安全性的金标准；然而，它受到高成本和长时间的限制。真实世界数据（RWD）可为比较研究提供强大的数据基础，但质量参差不齐。本综述介绍了目标试验模拟，即研究人员利用真实世界数据并遵循临床试验设计，预先定义暴露和结局，设定纳入标准，确定时间零点，估计样本量，并规划统计分析，以提高观察性研究的证据质量。本综述初步探讨了目标试验模拟中证据质量评估的标准。然后，通过案例解读展示目标试验模拟。
