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Review
. 2023 Sep 1;34(5):627-636.
doi: 10.1097/EDE.0000000000001633. Epub 2023 May 26.

An Overview of Current Methods for Real-world Applications to Generalize or Transport Clinical Trial Findings to Target Populations of Interest

Albee Y Ling  1 , Maria E Montez-Rath  2 , Paulo Carita  3 , Karen J Chandross  4 , Laurence Lucats  3 , Zhaoling Meng  5 , Bernard Sebastien  3 , Kris Kapphahn  1 , Manisha Desai  1
Affiliations
Review

An Overview of Current Methods for Real-world Applications to Generalize or Transport Clinical Trial Findings to Target Populations of Interest

Albee Y Ling et al. Epidemiology. .

Abstract

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.

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Figures

FIGURE.
FIGURE.
Sources of bias—measured or unmeasured—that threaten internal and external validity of a clinical study.
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