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We propose an information borrowing strategy for the design and monitoring of phase II basket trials based on the local multisource exchangeability assumption between baskets (disease types). We construct a flexible statistical design using the proposed strategy. Our approach partitions potentially heterogeneous baskets into non-exchangeable blocks. Information borrowing is only allowed to occur locally, i.e., among similar baskets within the same block. The amount of borrowing is determined by between-basket similarities. The number of blocks and block memberships are inferred from data based on the posterior probability of each partition. The proposed method is compared to the multisource exchangeability model and Simons two-stage design, respectively. In a variety of simulation scenarios, we demonstrate the proposed method is able to maintain the type I error rate and have desirable basket-wise power. In addition, our method is computationally efficient compared to existing Bayesian methods in that the posterior profiles of interest can be derived explicitly without the need for sampling algorithms.
Tissue-agnostic trials enroll patients based on their genetic biomarkers, not tumor type, in an attempt to determine if a new drug can successfully treat disease conditions based on biomarkers. The Bayesian hierarchical model (BHM) provides an attrac
We propose BaySize, a sample size calculator for phase I clinical trials using Bayesian models. BaySize applies the concept of effect size in dose finding, assuming the MTD is defined based on an equivalence interval. Leveraging a decision framework
Integrated phase I-II clinical trial designs are efficient approaches to accelerate drug development. In cases where efficacy cannot be ascertained in a short period of time, two-stage approaches are usually employed. When different patient populatio
Incorporating preclinical animal data, which can be regarded as a special kind of historical data, into phase I clinical trials can improve decision making when very little about human toxicity is known. In this paper, we develop a robust hierarchica
External pilot trials of complex interventions are used to help determine if and how a confirmatory trial should be undertaken, providing estimates of parameters such as recruitment, retention and adherence rates. The decision to progress to the conf