Performance and Sample Size Requirements of Bayesian Methods for Binary Outcomes in Fixed-Dose Combination Drug Studies

Abstract

We develop a Bayesian analysis for the study of fixed-dose combinations of two or more drugs. The approach described here does not require knowledge of the dose-response relationships of the components or large sample approximations. We provide a procedure to estimate sample size in this context. In addition, we explore the performance of the Bayesian procedure in situations where existing methods are known to perform poorly.

Key Words:

• Beta-binomial
• Power
• Sample size
• Synergy

ACKNOWLEDGMENT

We wish to thank the anonymous referees for their insightful comments that significantly improved this paper.

Notes

¹Programs used for the calculations herein may be obtained from the authors.