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Bayesian Phase II Single-Arm Designs

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Frontiers of Biostatistical Methods and Applications in Clinical Oncology

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Abstract

Phase II exploratory clinical trials in oncology are often designed as single-arm trials using a binary efficacy outcome with or without interim monitoring. This chapter focused on the Bayesian designs which considered both efficacy and safety outcomes associated with early stopping. In particular, we introduce a Bayesian adaptive design denoted as predictive sample size selection design (PSSD). The design allows for sample size selection following any planned interim analyses for early stopping of a trial, together with sample size determination. An extension of the PSSD to add continuous monitoring of safety is also described. We investigate the operating characteristics of the design through simulation studies.

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Acknowledgements

This work was supported in part by Grant-in Aid for Scientific Research (26330037) from Japan Society for the Promotion of Science, Japan. The author is grateful to Dr. Takashi Daimon, Hyogo College of Medicine, Hyogo, Japan, and Dr. Sarah Zohar, INSERM, U1138 team 22, Paris, France, for their contributions to this research.

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Authors and Affiliations

  1. Department of Biostatistics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, 465 Kajii-cho, Kawaramachi-Hirokoji, Kamigyo-ku, Kyoto, 602-8566, Japan

    Satoshi Teramukai

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  1. Satoshi Teramukai
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Correspondence to Satoshi Teramukai .

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Editors and Affiliations

  1. Graduate School of Medicine, Nagoya University Graduate School of Medicine, Nagoya, Aichi, Japan

    Shigeyuki Matsui

  2. Cancer Research and Biostatistics, Seattle, Washington, USA

    John Crowley

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Teramukai, S. (2017). Bayesian Phase II Single-Arm Designs. In: Matsui, S., Crowley, J. (eds) Frontiers of Biostatistical Methods and Applications in Clinical Oncology. Springer, Singapore. https://doi.org/10.1007/978-981-10-0126-0_6

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