Considerations for the Integration of Clinical Trials and Real-World Data.

Authors

Sky Qiu, Charles E Barr, Lauren Dang, Larry Han, Kajsa Kvist, Hana Lee, Andrew Mertens, Nerissa Nance, Lei Nie, Kara Rudolph, Xu Shi, Jens Tarp, Salina P Waddy, Kenneth Wiley, Andy Wilson, Margot Lisa Jing Yann, Zhiwei Zhang, Tianyue Zhou, Maya Petersen, Mark van der Laan

Published in

Clinical pharmacology and therapeutics. Oct 05, 2026. Epub Oct 05, 2026.

Abstract

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of randomized controlled trials and real-world data, grounded in explicit causal frameworks, offers a path toward evidence that is both internally credible and externally relevant. In this article, we describe distinct objectives for the integration of randomized controlled trials and real-world data and discuss how these objectives shape key design and analytic considerations, illustrating the resulting choices through example estimands. We highlight practical issues that commonly arise in applied settings, including data relevance and curation, cross-source comparability, estimand specification, and sensitivity analysis. We aim for this article to help readers evaluate and implement principled approaches to integrating randomized controlled trials and real-world data in ways that can support more reliable treatment recommendations while maintaining regulatory-grade evidentiary standards.

PMID:
42834704
Bibliographic data and abstract were imported from PubMed on 06 Oct 2026.

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