Bayesian uncertainty directed trial designs

DOI10.1080/01621459.2018.1497497MaRDI QIDQ5242442zbMATH OpenOpenAlexWikidataFDO

Authors Steffen Ventz, Matteo Cellamare, Sergio Bacallado, Lorenzo Trippa

Publication date 12 November 2019

Published in Journal of the American Statistical Association (Search for Journal in Brave)

Full work available at URL https://arxiv.org/abs/1806.11370

zbMATH Keywords

decision theoryinformation theorymulti-arm clinical trialsresponse-adaptive designs

Mathematics Subject Classification ID

Sampling theory, sample surveys (62D05) Bayesian inference (62F15) Fuzziness and design of statistical experiments (62K86) Applications of statistics to biology and medical sciences; meta analysis (62P10)

Abstract: Most Bayesian response-adaptive designs unbalance randomization rates towards the most promising arms with the goal of increasing the number of positive treatment outcomes during the study, even though the primary aim of the trial is different. We discuss Bayesian uncertainty directed designs (BUD), a class of Bayesian designs in which the investigator specifies an information measure tailored to the experiment. All decisions during the trial are selected to optimize the available information at the end of the study. The approach can be applied to several designs, ranging from early stage multi-arm trials to biomarker-driven and multi-endpoint studies. We discuss the asymptotic limit of the patient allocation proportion to treatments, and illustrate the finite-sample operating characteristics of BUD designs through examples, including multi-arm trials, biomarker-stratified trials, and trials with multiple co-primary endpoints.

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Cited in

(4)

• Approximating the operating characteristics of Bayesian uncertainty directed trial designs
• Estimating design operating characteristics in Bayesian adaptive clinical trials
• Comment to: ``Response-adaptive randomization in clinical trials: from myths to practical considerations
• Bayesian adaptive \(N\)-of-1 trials for estimating population and individual treatment effects

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