Hybrid classical-Bayesian approach to sample size determination for two-arm superiority clinical trials

Sambucini Valeria ()
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Sambucini Valeria: Dipartimento di Scienze Statistiche, Sapienza University of Rome, Piazzale Aldo Moro n. 5, 00185 Roma, Italy

The International Journal of Biostatistics, 2024, vol. 20, issue 2, 553-570

Abstract: Traditional methods for Sample Size Determination (SSD) based on power analysis exploit relevant fixed values or preliminary estimates for the unknown parameters. A hybrid classical-Bayesian approach can be used to formally incorporate information or model uncertainty on unknown quantities by using prior distributions according to the Bayesian approach, while still analysing the data in a frequentist framework. In this paper, we propose a hybrid procedure for SSD in two-arm superiority trials, that takes into account the different role played by the unknown parameters involved in the statistical power. Thus, different prior distributions are used to formalize design expectations and to model information or uncertainty on preliminary estimates involved at the analysis stage. To illustrate the method, we consider binary data and derive the proposed hybrid criteria using three possible parameters of interest, i.e. the difference between proportions of successes, the logarithm of the relative risk and the logarithm of the odds ratio. Numerical examples taken from the literature are presented to show how to implement the proposed procedure.

Keywords: binary data; conditional approach; hybrid classical-Bayesian; sample size determination; unconditional approach (search for similar items in EconPapers)
Date: 2024
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DOI: 10.1515/ijb-2023-0050

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