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.
Authors
- Jianrong Wu (ORCID: https://orcid.org/0009-0004-7088-1373)
- Yuntong Li (ORCID: https://orcid.org/0000-0002-3071-4333)
Institutions
- University of New Mexico (US)
- Regeneron (United States) (US)
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
Funders
- National Cancer Institute