Kim Tae-Eun, Park Sang-In, Shin Kwang-Hee
Department of Clinical Pharmacology, Konkuk University Medical Center, Seoul 05030, Korea.
Department of Pharmacology, College of Medicine, Kangwon National University, Chuncheon 24341, Korea.
Transl Clin Pharmacol. 2022 Sep;30(3):121-128. doi: 10.12793/tcp.2022.30.e14. Epub 2022 Sep 15.
As real-world data (RWD) becomes more available and the methodology for handling RWD evolves, the use of RWD in drug development and drug approval is drawing interest. One of the ways RWD can be applied to a clinical trial is using an external control, a cohort of patients established separately serving as a control group for the clinical trial's treatment group. Although external controls have the possibility of bias as a result of differences in baseline characteristics between the external control and experimental groups, selecting an appropriate data source and ensuring comparability through proper handling of the data can increase the utility of external controls, raising the efficiency of drug development. This article discusses several topics relevant to using external controls in clinical trials, including the definition of external control, the selection of data sources, the strategy ensuring comparability, current regulatory circumstances, and future directions.
随着真实世界数据（RWD）越来越容易获取，且处理RWD的方法不断发展，RWD在药物研发和药物审批中的应用正引起人们的关注。RWD应用于临床试验的一种方式是使用外部对照，即单独设立的一组患者作为临床试验治疗组的对照组。尽管由于外部对照组与实验组之间基线特征存在差异，外部对照存在偏倚的可能性，但选择合适的数据源并通过对数据的适当处理确保可比性，可以提高外部对照的效用，从而提高药物研发的效率。本文讨论了与在临床试验中使用外部对照相关的几个主题，包括外部对照的定义、数据源的选择、确保可比性的策略、当前的监管情况以及未来的发展方向。
