Bayesian Approaches to Subgroup Analysis and Related Adaptive Clinical Trial Designs.

We review Bayesian and Bayesian decision theoretic approaches to subgroup analysis and applications to subgroup-based adaptive clinical trial designs. Subgroup analysis refers to inference about subpopulations with significantly distinct treatment effects. The discussion mainly focuses on inference for a benefiting subpopulation, that is, a characterization of a group of patients who benefit from the treatment under consideration more than the overall population. We introduce alternative approaches and demonstrate them with a small simulation study. Then, we turn to clinical trial designs. When the selection of the interesting subpopulation is carried out as the trial proceeds, the design becomes an adaptive clinical trial design, using subgroup analysis to inform the randomization and assignment of treatments to patients. We briefly review some related designs. There are a variety of approaches to Bayesian subgroup analysis. Practitioners should consider the type of subpopulations in which they are interested and choose their methods accordingly. We demonstrate how subgroup analysis can be carried out by different Bayesian methods and discuss how they identify slightly different subpopulations.