The New England Journal of Statistics in Data Science

Basket trials have captured much attention in oncology research in recent years, as advances in health technology have opened up the possibility of classification of patients at the genomic level. Bayesian methods are particularly prevalent in basket trials as the hierarchical structure is adapted to basket trials to allow for information borrowing. In this article, we extend the Bayesian methods to basket trials with treatment and control arms for continuous endpoints, which are often the cases in clinical trials for rare diseases. To account for the imbalance in the covariates which are potentially strong predictors but not stratified in a randomized trial, our models make adjustments for these covariates, and allow different coefficients across baskets. In addition, comparisons are drawn between two-stage design and one-stage design for the four Bayesian methods. Extensive simulation studies are conducted to examine the empirical performance of all models under consideration. A real data analysis is carried out to further demonstrate the usefulness of the Bayesian methods.