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Bayesian single-arm phase II trial designs with time-to-event endpoints
Jianrong Wu1, Haitao Pan2, Chia-Wei Hsu2
1Biostatistics and Bioinformatics Shared Resource Facility, Markey Cancer Center, University of Kentucky, Lexington, Kentucky, USA.
Pharmaceutical Statistics
|June 4, 2021
Summary
This study introduces novel Bayesian trial designs for cancer research, focusing on time-to-event endpoints in immunotherapy and targeted therapy trials. These methods offer practical tools for designing efficient clinical trials with robust statistical properties.
Area of Science:
- Biostatistics
- Clinical Trial Design
- Oncology
Background:
- Time-to-event endpoints are crucial in cancer clinical trials, particularly for immunotherapy and molecularly targeted therapies.
- Bayesian approaches offer advantages for single-arm Phase II trial designs, especially with small sample sizes.
Purpose of the Study:
- To present an event-driven Bayesian approach for one-stage and two-stage single-arm Phase II trial designs.
- To develop theoretical relationships between frequentist and Bayesian trial designs.
- To provide practical tools for investigators designing cancer clinical trials.
Main Methods:
- Developed Bayesian one-stage and two-stage trial designs.
- Established theoretical links between frequentist and Bayesian design properties.
- Utilized exact posterior distributions for small sample sizes.
- Extended Simon's two-stage design for time-to-event endpoints.
Main Results:
- Proposed executable algorithms for Bayesian one-stage designs.
- Demonstrated how frequentist properties can be achieved with Bayesian designs.
- Showcased the accommodation of small sample sizes in Phase II trials.
- Comprehensive simulations evaluated the frequentist properties of the proposed Bayesian designs.
Conclusions:
- The developed Bayesian designs provide a flexible and statistically sound framework for cancer clinical trials.
- An R package, BayesDesign, is available for convenient implementation of these methods.
- These advancements facilitate the design of efficient Phase II trials with time-to-event endpoints.
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