Estimating Subgroup Effects in Generalizability and Transportability Analyses

Am J Epidemiol. 2024 Jan 8;193(1):149-158. doi: 10.1093/aje/kwac036.


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 nonrandomized 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 nonrandomized subset, and we 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 (North America, 1975-1996) 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.

Keywords: generalizability; heterogeneity of treatment effects; subgroup analysis; transportability.

MeSH terms

  • Humans
  • Myocardial Infarction* / therapy
  • North America