A Critical Review of Methods for Real-World Applications to Generalize or Transport Clinical Trial Findings to Target Populations of Interest

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...

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Bibliographic Details
Published in:arXiv.org 2022-02
Main Authors: Ling, Albee Y, Montez-Rath, Maria E, Carita, Paulo, Chandross, Karen, Lucats, Laurence, Meng, Zhaoling, Bernard, Sebastien, Kapphahn, Kris, Desai, Manisha
Format: Article
Language:English
Subjects:
Populations
State-of-the-art reviews
Online Access:Get full text
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Summary: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.
ISSN:2331-8422