Generalizing causal inferences from individuals in randomized trials to all trial-eligible individuals

DOI10.1111/BIOM.13009MaRDI QIDQ5214531zbMATH OpenOpenAlexWikidataFDO

Authors Issa J. Dahabreh, Sarah E. Robertson, Eric J. Tchetgen Tchetgen, Elizabeth A. Stuart, Miguel A. Hernán

Publication date 7 February 2020

Published in Biometrics (Search for Journal in Brave)

Full work available at URL https://arxiv.org/abs/1709.04589

zbMATH Keywords

causal inferenceclinical trialsdouble robustnessobservational studiesgeneralizabilitytransportability

Mathematics Subject Classification ID

Applications of statistics to biology and medical sciences; meta analysis (62P10) Sampling theory, sample surveys (62D05)

Abstract: We consider methods for causal inference in randomized trials nested within cohorts of trial-eligible individuals, including those who are not randomized. We show how baseline covariate data from the entire cohort, and treatment and outcome data only from randomized individuals, can be used to identify potential (counterfactual) outcome means and average treatment effects in the target population of all eligible individuals. We review identifiability conditions, propose estimators, and assess the estimators' finite-sample performance in simulation studies. As an illustration, we apply the estimators in a trial nested within a cohort of trial-eligible individuals to compare coronary artery bypass grafting surgery plus medical therapy vs. medical therapy alone for chronic coronary artery disease.

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Cited in

(13)

• Estimating the Area under the ROC Curve When Transporting a Prediction Model to a Target Population
• A note on semiparametric efficient generalization of causal effects from randomized trials to target populations
• Sensitivity analysis for an unobserved moderator in RCT-to-target-population generalization of treatment effects
• Data integration in causal inference
• Efficient and Robust Methods for Causally Interpretable Meta-Analysis: Transporting Inferences from Multiple Randomized Trials to a Target Population
• Leveraging population outcomes to improve the generalization of experimental results: application to the JTPA study
• Transfer Learning of Individualized Treatment Rules from Experimental to Real-World Data
• Comment on: ``Target estimands for population-adjusted indirect comparisons
• Generalizing the Intention-to-Treat Effect of an Active Control from Historical Placebo-Controlled Trials: A Case Study of the Efficacy of Daily Oral TDF/FTC in the HPTN 084 Study
• Improving Trial Generalizability Using Observational Studies
• Causal inference methods for combining randomized trials and observational studies: a review
• Extending inferences from a randomized trial to a new target population
• Sensitivity analysis using bias functions for studies extending inferences from a randomized trial to a target population

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