Abstract
Compared to single-arm phase II trials, randomized phase II trials provide a more reliable way to assess the efficacy of experimental drugs and make the Go/No-Go decision. This chapter introduces a frequentist design, followed by several Bayesian randomized phase II designs, including randomized Bayesian optimal phase 2 (BOP2) design and two-stage screened selection design. This chapter focuses on Bayesian designs because of their flexibility and efficiency to handle the complexity of immunotherapies and targeted therapies that may involve binary, survival, ordinal, co-primary and multiple endpoints. A two-stage screened selection design (SSD) for randomized trials but without the control treatment is also introduced. Software to implement these designs are described.
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Notes
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Pan, H., Yuan, Y. (2023). Randomized Phase II Designs. In: Bayesian Adaptive Design for Immunotherapy and Targeted Therapy. Springer, Singapore. https://doi.org/10.1007/978-981-19-8176-0_7
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DOI: https://doi.org/10.1007/978-981-19-8176-0_7
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