Estimation of treatment effect in a subpopulation: An empirical Bayes approach

ABSTRACT

It is well recognized that the benefit of a medical intervention may not be distributed evenly in the target population due to patient heterogeneity, and conclusions based on conventional randomized clinical trials may not apply to every person. Given the increasing cost of randomized trials and difficulties in recruiting patients, there is a strong need to develop analytical approaches to estimate treatment effect in subpopulations. In particular, due to limited sample size for subpopulations and the need for multiple comparisons, standard analysis tends to yield wide confidence intervals of the treatment effect that are often noninformative. We propose an empirical Bayes approach to combine both information embedded in a target subpopulation and information from other subjects to construct confidence intervals of the treatment effect. The method is appealing in its simplicity and tangibility in characterizing the uncertainty about the true treatment effect. Simulation studies and a real data analysis are presented.

KEYWORDS:

• Causal inference
• empirical bayes
• heterogeneity in treatment effect
• subgroup analysis

Disclaimer

This manuscript was prepared using MAGIC research materials obtained from the National Heart, Lung and Blood Institute (NHLBI) Biologic Specimen and Data Repository Information Coordinating Center and does not necessarily reflect the opinions or views of the MAGIC or the NHLBI.

Funding

This work is supported in part by National Institutes of Health (NIH) grant R21 CA152463 and the Indiana University Health–Indiana University School of Medicine Strategic Research Initiative in Cardiology.

Additional information

Funding

This work is supported in part by National Institutes of Health (NIH) grant R21 CA152463 and the Indiana University Health–Indiana University School of Medicine Strategic Research Initiative in Cardiology.