Bayesian optimal Phase II survival trial design with event-driven approach

Bayesian design incorporates prior knowledge and external information, making it an attractive option during the early phase of a clinical trial. A number of Bayesian optimal designs have been proposed to make go/no-go decisions based on posterior probabilities while also having desired frequentist operating characteristics. However, existing Bayesian designs either are not appropriate for time-to-event endpoints or rely on an exponential distribution assumption on the data. In this paper, we propose a Bayesian optimal Phase II event-driven design (BOP2e) that allows for futility and/or superiority stopping for single-arm trials with a time-to-event endpoint. The proposed BOP2e design is optimal in minimizing the expected sample size under null hypothesis while also controlling the frequentist Type I error. Simulation studies are performed to explore the operating characteristics of the proposed BOP2e designs. A user-friendly Shiny application is available to help implement the proposed designs.

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Publication Details

Journal
Journal of Biopharmaceutical Statistics
Published
2025-06-08
DOI
https://doi.org/10.1080/10543406.2025.2512202
Primary Topic
Statistical Methods in Clinical Trials
Type
article
Field-Weighted Citation Impact
0.00

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article

Bayesian optimal Phase II survival trial design with event-driven approach

Jianrong Wu, Yuntong Li
Journal of Biopharmaceutical Statistics
Statistical Methods in Clinical Trials
article

Bayesian optimal Phase II survival trial design with event-driven approach

Jianrong Wu, Yuntong Li
article en

Abstract

Bayesian design incorporates prior knowledge and external information, making it an attractive option during the early phase of a clinical trial. A number of Bayesian optimal designs have been proposed to make go/no-go decisions based on posterior probabilities while also having desired frequentist operating characteristics. However, existing Bayesian designs either are not appropriate for time-to-event endpoints or rely on an exponential distribution assumption on the data. In this paper, we propose a Bayesian optimal Phase II event-driven design (BOP2e) that allows for futility and/or superiority stopping for single-arm trials with a time-to-event endpoint. The proposed BOP2e design is optimal in minimizing the expected sample size under null hypothesis while also controlling the frequentist Type I error. Simulation studies are performed to explore the operating characteristics of the proposed BOP2e designs. A user-friendly Shiny application is available to help implement the proposed designs.

Journal of Biopharmaceutical Statistics
University of New Mexico (US), Regeneron (United States) (US)
National Cancer Institute
Climate action
Openalex Percentile: Top 87%
Statistical Methods in Clinical Trials
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Bayesian optimal Phase II survival trial design with event-driven approach — Jianrong Wu, Yuntong Li · Journal of Biopharmaceutical Statistics (2025) | TGRS Research Map | TGRS