2019/05/17 by Xiao Wu, Wu, Xiao, Y. Jun Xu +3
Decision Sciences · Economics, Econometrics and Finance · Mathematics · #Applications (stat.AP) #Computation (stat.CO) #FOS: Computer and information sciences #Health Systems, Economic Evaluations, Quality of Life #Methodology (stat.ME) #Optimal Experimental Design Methods #Statistical Methods in Clinical Trials
paper · pdf · doi:10.48550/arxiv.1905.07456
openalex publication_date 2019/05/17 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/28
In developing products for rare diseases, statistical challenges arise due to the limited number of patients available for participation in drug trials and other clinical research. Bayesian adaptive clinical trial designs offer the possibility of increased statistical efficiency, reduced development cost and ethical hazard prevention via their incorporation of evidence from external sources (historical data, expert opinions, and real-world evidence), and flexibility in the specification of interim looks. In this paper, we propose a novel Bayesian adaptive commensurate design that borrows adaptively from historical information and also uses a particular payoff function to optimize the timing of the study's interim analysis. The trial payoff is a function of how many samples can be saved via early stopping and the probability of making correct early decisions for either futility or efficacy. We calibrate our Bayesian algorithm to have acceptable long-run frequentist properties (Type I error and power) via simulation at the design stage. We illustrate our approach using a pediatric trial design setting testing the effect of a new drug for a rare genetic disease. The optimIA R package available at https://github.com/wxwx1993/BayesianIATiming provides an easy-to-use implementation of our approach.