Efficacy and toxicity monitoring via Bayesian predictive probabilities in phase II clinical trials

Valeria Sambucini ()
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Valeria Sambucini: Università degli Studi di Roma La Sapienza

Statistical Methods & Applications, 2021, vol. 30, issue 2, No 10, 637-663

Abstract: Abstract Bayesian monitoring strategies based on predictive probabilities are widely used in phase II clinical trials that involve a single efficacy binary variable. The essential idea is to control the predictive probability that the trial will show a conclusive result at the scheduled end of the study, given the information at the interim stage and the prior beliefs. In this paper, we present an extension of this approach to incorporate toxicity considerations in single-arm phase II trials. We consider two binary endpoints representing response and toxicity of the experimental treatment and define the result as successful at the conclusion of the study if the posterior probability of an high efficacy and that of a small toxicity are both sufficiently large. At any interim look, the Multinomial-Dirichlet distribution provides the predictive probability of each possible combination of future efficacy and toxicity outcomes. It is exploited to obtain the predictive probability that the trial will yield a positive outcome, if it continues to the planned end. Different possible interim situations are considered to investigate the behaviour of the proposed predictive rules and the differences with the monitoring strategies based on posterior probabilities are highlighted. Simulation studies are also performed to evaluate the frequentist operating characteristics of the proposed design and to calibrate the design parameters.

Keywords: Bayesian approach; Efficacy and toxicity outcomes; Multinomial-Dirichlet distribution; Posterior predictive probabilities; Single-arm phase II trials (search for similar items in EconPapers)
Date: 2021
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DOI: 10.1007/s10260-020-00537-3

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