Estimating subgroup effects in generalizability and transportability analyses

Author:

Robertson Sarah E12,Steingrimsson Jon A3,Joyce Nina R4,Stuart Elizabeth A5,Dahabreh Issa J16

Affiliation:

1. CAUSALab, Harvard T.H. Chan School of Public Health, Boston, MA

2. Department of Health Services, Policy & Practice, Brown University School of Public Health, Providence, RI

3. Department of Biostatistics, Brown University School of Public Health, Providence, RI

4. Department of Epidemiology, Brown University School of Public Health, Providence, RI

5. Departments of Mental Health, Biostatistics, and Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD

6. Departments of Epidemiology and Biostatistics, Harvard T.H. Chan School of Public Health, Boston, MA

Abstract

Abstract Methods for extending – generalizing or transporting – inferences from a randomized trial to a target population involve conditioning on a large set of covariates that is sufficient for rendering the randomized and non-randomized groups exchangeable. Yet, decision-makers are often interested in examining treatment effects in subgroups of the target population defined in terms of only a few discrete covariates. Here, we propose methods for estimating subgroup-specific potential outcome means and average treatment effects in generalizability and transportability analyses, using outcome model-based (g-formula), weighting, and augmented weighting estimators. We consider estimating subgroup-specific average treatment effects in the target population and its non-randomized subset, and provide methods that are appropriate both for nested and non-nested trial designs. As an illustration, we apply the methods to data from the Coronary Artery Surgery Study to compare the effect of surgery plus medical therapy versus medical therapy alone for chronic coronary artery disease in subgroups defined by history of myocardial infarction.

Publisher

Oxford University Press (OUP)

Subject

Epidemiology

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