TY - JOUR
T1 - Assessing Heterogeneity of Treatment Effect in Real-World Data
AU - Segal, Jodi B.
AU - Varadhan, Ravi
AU - Groenwold, Rolf H.H.
AU - Henderson, Nicholas C.
AU - Li, Xiaojuan
AU - Nomura, Kaori
AU - Kaplan, Sigal
AU - Ardeshirrouhanifard, Shirin
AU - Heyward, James
AU - Nyberg, Fredrik
AU - Burcu, Mehmet
N1 - Funding Information:
Grant Support: Dr. Varadhan's work was supported through Regional Oncology Research Center grant P30CA006973 from the National Cancer Institute.
Publisher Copyright:
© 2023 American College of Physicians.
PY - 2023/4/1
Y1 - 2023/4/1
N2 - 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.
AB - 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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U2 - 10.7326/M22-1510
DO - 10.7326/M22-1510
M3 - Article
C2 - 36940440
AN - SCOPUS:85152623572
SN - 0003-4819
VL - 176
SP - 536
EP - 544
JO - Annals of internal medicine
JF - Annals of internal medicine
IS - 4
ER -