A Bayesian approach to study design and analysis with type I error rate control for response variables of mixed types

Abstract

There has been increased interest in the design and analysis of studies consisting of multiple response variables of mixed types. For example, in clinical trials, it is desirable to establish efficacy for a treatment effect in primary and secondary outcomes. In this article, we develop Bayesian approaches for hypothesis testing and study planning for data consisting of multiple response variables of mixed types with covariates. We assume that the responses are correlated via a Gaussian copula, and that the model for each response is, marginally, a generalized linear model (GLM). Taking a fully Bayesian approach, the proposed method enables inference based on the joint posterior distribution of the parameters. Under some mild conditions, we show that the joint distribution of the posterior probabilities under any Bayesian analysis converges to a Gaussian copula distribution as the sample size tends to infinity. Using this result, we develop an approach to control the type I error rate under multiple testing. Simulation results indicate that the method is more powerful than conducting marginal regression models and correcting for multiplicity using the Bonferroni‐Holm Method. We also develop a Bayesian approach to sample size determination in the presence of response variables of mixed types, extending the concept of probability of success (POS) to multiple response variables of mixed types.