Bayesian single-arm phase II trial designs with time-to-event endpoints |
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Authors: | Jianrong Wu Haitao Pan Chia-Wei Hsu |
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Affiliation: | 1. Biostatistics and Bioinformatics Shared Resource Facility, Markey Cancer Center, University of Kentucky, Lexington, Kentucky, USA;2. Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA |
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Abstract: | For the cancer clinical trials with immunotherapy and molecularly targeted therapy, time-to-event endpoint is often a desired endpoint. In this paper, we present an event-driven approach for Bayesian one-stage and two-stage single-arm phase II trial designs. Two versions of Bayesian one-stage designs were proposed with executable algorithms and meanwhile, we also develop theoretical relationships between the frequentist and Bayesian designs. These findings help investigators who want to design a trial using Bayesian approach have an explicit understanding of how the frequentist properties can be achieved. Moreover, the proposed Bayesian designs using the exact posterior distributions accommodate the single-arm phase II trials with small sample sizes. We also proposed an optimal two-stage approach, which can be regarded as an extension of Simon's two-stage design with the time-to-event endpoint. Comprehensive simulations were conducted to explore the frequentist properties of the proposed Bayesian designs and an R package BayesDesign can be assessed via R CRAN for convenient use of the proposed methods. |
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Keywords: | Bayesian design phase II trial proportional hazards sample size calculation time-to-event endpoint |
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