From the Quantitative Sciences Unit, Division of Biomedical Informatics Research, Department of Medicine, Stanford University School of Medicine, Palo Alto, CA.
Division of Nephrology, Department of Medicine, Stanford University School of Medicine, Palo Alto, CA.
Epidemiology. 2023 Sep 1;34(5):627-636. doi: 10.1097/EDE.0000000000001633. Epub 2023 May 26.
It has been well established that randomized clinical trials have poor external validity, resulting in findings that may not apply to relevant-or target-populations. When the trial is sampled from the target population, generalizability methods have been proposed to address the applicability of trial findings to target populations. When the trial sample and target populations are distinct, transportability methods may be applied for this purpose. However, generalizability and transportability studies present challenges, particularly around the strength of their conclusions. We review and summarize state-of-the-art methods for translating trial findings to target populations. We additionally provide a novel step-by-step guide to address these challenges, illustrating principles through a published case study. When conducted with rigor, generalizability and transportability studies can play an integral role in regulatory decisions by providing key real-world evidence.
随机临床试验的外部有效性较差已经得到充分证实，导致研究结果可能不适用于相关或目标人群。当试验从目标人群中抽样时，已经提出了推广方法来解决试验结果在目标人群中的适用性问题。当试验样本和目标人群不同时，可以应用可转移性方法来达到此目的。然而，推广性和可转移性研究存在挑战，特别是在其结论的强度方面。我们回顾和总结了将试验结果转化为目标人群的最新方法。此外，我们还提供了一个新颖的分步指南来解决这些挑战，通过一个已发表的案例研究来说明这些原则。当严格进行时，推广性和可转移性研究可以通过提供关键的真实世界证据，在监管决策中发挥重要作用。
