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The Bayesian Group-Sequential Predictive Evidence Value Design for Phase II Clinical Trials with Binary Endpoints

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  • Riko Kelter

    (University of Siegen)

  • Alexander Schnurr

    (University of Siegen)

Abstract

In clinical research, the initial efficacy of a new agent is typically assessed in a phase IIA study. Bayesian group-sequential designs are often based on predictive probability of trial success. In this paper, the novel Bayesian group-sequential predictive evidence value design is introduced, and we prove that the predictive probability approach is a special case of it. A comparison with Simon’s two-stage and competing Bayesian designs based on phase IIA cancer trials is provided. Results show that the novel design can improve operating characteristics such as the false-positive rate, probability of early stopping for futility and expected sample size of the trial. Given these advantages, the predictive evidence value design constitutes an important addition to the biostatistician’s toolbelt when planning a phase IIA trial the Bayesian way, in particular, when small sample sizes and a large probability for early termination under the null hypothesis are desired.

Suggested Citation

  • Riko Kelter & Alexander Schnurr, 2025. "The Bayesian Group-Sequential Predictive Evidence Value Design for Phase II Clinical Trials with Binary Endpoints," Statistics in Biosciences, Springer;International Chinese Statistical Association, vol. 17(2), pages 442-478, July.
    • Handle: RePEc:spr:stabio:v:17:y:2025:i:2:d:10.1007_s12561-024-09430-z
    DOI: 10.1007/s12561-024-09430-z
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    References listed on IDEAS

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    1. Alessandra Giovagnoli, 2021. "The Bayesian Design of Adaptive Clinical Trials," IJERPH, MDPI, vol. 18(2), pages 1-15, January.
    2. Koehler, Elizabeth & Brown, Elizabeth & Haneuse, Sebastien J.-P. A., 2009. "On the Assessment of Monte Carlo Error in Simulation-Based Statistical Analyses," The American Statistician, American Statistical Association, vol. 63(2), pages 155-162.
    3. Alexander Shapiro & Jos Berge, 2002. "Statistical inference of minimum rank factor analysis," Psychometrika, Springer;The Psychometric Society, vol. 67(1), pages 79-94, March.
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