Robust Bayesian sample size determination in clinical trials

Statistics in Medicine
Pierpaolo BruttiStefania Gubbiotti

Abstract

This article deals with determination of a sample size that guarantees the success of a trial. We follow a Bayesian approach and we say an experiment is successful if it yields a large posterior probability that an unknown parameter of interest (an unknown treatment effect or an effects-difference) is greater than a chosen threshold. In this context, a straightforward sample size criterion is to select the minimal number of observations so that the predictive probability of a successful trial is sufficiently large. In the paper we address the most typical criticism to Bayesian methods-their sensitivity to prior assumptions-by proposing a robust version of this sample size criterion. Specifically, instead of a single distribution, we consider a class of plausible priors for the parameter of interest. Robust sample sizes are then selected by looking at the predictive distribution of the lower bound of the posterior probability that the unknown parameter is greater than a chosen threshold. For their flexibility and mathematical tractability, we consider classes of epsilon-contamination priors. As specific applications we consider sample size determination for a Phase III trial.

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Citations

Jun 22, 2013·Journal of Biopharmaceutical Statistics·James D StameyJohn W Seaman
Feb 19, 2016·Journal of Biopharmaceutical Statistics·Paul FayaJames D Stamey
May 23, 2009·Statistics in Medicine·Pierpaolo BruttiStefania Gubbiotti
Jun 20, 2012·Statistics in Medicine·J Jack Lee, Caleb T Chu
Jul 19, 2012·Statistics in Medicine·Satoshi TeramukaiSarah Zohar
Nov 26, 2009·Biometrics·Bhramar MukherjeeNilanjan Chatterjee
Dec 30, 2014·Statistics in Medicine·Matteo Cellamare, Valeria Sambucini
Jul 31, 2013·Pharmaceutical Statistics·Karen L PriceLaura Thompson
Oct 15, 2013·Statistics in Medicine·A Lawrence Gould, Xiaohua Douglas Zhang
Feb 20, 2016·Journal of Biopharmaceutical Statistics·Pierpaolo BruttiValeria Sambucini
Feb 7, 2017·Journal of Biopharmaceutical Statistics·Erik PulkstenisJianliang Zhang
Jun 6, 2018·Statistical Methods in Medical Research·Maria M Ciarleglio, Christopher D Arendt
Jul 13, 2019·Statistics in Medicine·Lawrence Joseph, Patrick Bélisle
Jan 16, 2021·International Journal of Environmental Research and Public Health·Fulvio De Santis, Stefania Gubbiotti

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