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Assessing Heterogeneity of Treatment Effect in Real-World Data

ARTICLE: Assessing Heterogeneity of Treatment Effect in Real-World Data

AUTHORS: Jodi B Segal, Ravi Varadhan, Rolf H H Groenwold, Xiaojuan Li, Kaori Nomura, Sigal Kaplan, Shirin Ardeshirrouhanifard, James Heyward, Fredrik Nyberg, Mehmet Burcu

JOURNAL: Ann Intern Med. 2023 Apr;176(4):536-544. doi: 10.7326/M22-1510. Epub 2023 Mar 21.


Increasing availability of real-world data (RWD) generated from patient care enables the generation of evidence to inform clinical decisions for subpopulations of patients and perhaps even individuals. There is growing opportunity to identify important heterogeneity of treatment effects (HTE) in these subgroups. Thus, HTE is relevant to all with interest in patients' responses to interventions, including regulators who must make decisions about products when signals of harms arise postapproval and payers who make coverage decisions based on expected net benefit to their beneficiaries. Prior work discussed HTE in randomized studies. Here, we address methodological considerations when investigating HTE in observational studies. We propose 4 primary goals of HTE analyses and the corresponding approaches in the context of RWD: to confirm subgroup effects, to describe the magnitude of HTE, to discover clinically important subgroups, and to predict individual effects. We discuss other possible goals including exploring prognostic score- and propensity score-based treatment effects, and testing the transportability of trial results to populations different from trial participants. Finally, we outline methodological needs for enhancing real-world HTE analysis.

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Kelsey Bennett