论文标题
对现实世界应用的方法的批判性综述，以概括或运输临床试验结果针对目标人群
A Critical Review of Methods for Real-World Applications to Generalize or Transport Clinical Trial Findings to Target Populations of Interest
论文作者
论文摘要
已经提出了可推广性和可运输方法来解决随机临床试验的外部有效性偏差，该试验是由于试验和目标人群之间治疗效应改性剂的分布差异而导致的。但是，这样的研究提出了许多挑战。我们审查并总结了最先进的方法论方面的考虑。我们还为调查人员提供了一份逐步指南，以解决这些挑战，并通过公开的案例研究说明。当严格进行时，此类研究可能通过提供关键的现实证据来在监管决策中起着不可或缺的作用。
Generalizability and transportability methods have been proposed to address the external validity bias of randomized clinical trials that results from differences in the distribution of treatment effect modifiers between trial and target populations. However, such studies present many challenges. We review and summarize state-of-the-art methodological considerations. We additionally provide investigators with a step-by-step guide to address these challenges, illustrated through a published case study. When conducted with rigor, such studies may play an integral role in regulatory decisions by providing key real-world evidence.
