Frequentist and Bayesian Sample Size Determination for Single-Arm Clinical Trials Based on a Binary Response Variable: A Shiny App to Implement Exact Methods  

作　　者：Susanna Gentile Valeria Sambucini Susanna Gentile;Valeria Sambucini(Department of Statistical Sciences, Sapienza University of Rome, Rome, Italy)

机构地区：[1]Department of Statistical Sciences, Sapienza University of Rome, Rome, Italy

出　　处：《Open Journal of Statistics》2024年第1期90-105,共16页统计学期刊（英文）

摘　　要：Sample size determination typically relies on a power analysis based on a frequentist conditional approach. This latter can be seen as a particular case of the two-priors approach, which allows to build four distinct power functions to select the optimal sample size. We revise this approach when the focus is on testing a single binomial proportion. We consider exact methods and introduce a conservative criterion to account for the typical non-monotonic behavior of the power functions, when dealing with discrete data. The main purpose of this paper is to present a Shiny App providing a user-friendly, interactive tool to apply these criteria. The app also provides specific tools to elicit the analysis and the design prior distributions, which are the core of the two-priors approach.Sample size determination typically relies on a power analysis based on a frequentist conditional approach. This latter can be seen as a particular case of the two-priors approach, which allows to build four distinct power functions to select the optimal sample size. We revise this approach when the focus is on testing a single binomial proportion. We consider exact methods and introduce a conservative criterion to account for the typical non-monotonic behavior of the power functions, when dealing with discrete data. The main purpose of this paper is to present a Shiny App providing a user-friendly, interactive tool to apply these criteria. The app also provides specific tools to elicit the analysis and the design prior distributions, which are the core of the two-priors approach.

关 键 词：Binomial Proportion Frequentist and Bayesian Power Functions Exact Sample Size Determination Shiny App Two-Priors Approach 

分 类 号：TP3[自动化与计算机技术—计算机科学与技术]