The distribution of power-related random variables (and their use in clinical trials)

Abstract In the hybrid Bayesian-frequentist approach to hypotheses tests, the power function, i.e. the probability of rejecting the null hypothesis, is a random variable and a pre-experimental evaluation of the study is commonly carried out through the so-called probability of success (PoS). PoS is usually defined as the expected value of the random power that is not necessarily a well-representative summary of the entire distribution. Here, we consider the main definitions of PoS and investigate the power related random variables that induce them. We provide general expressions for their cumulative distribution and probability density functions, as well as closed-form expressions when the test statistic is, at least asymptotically, normal. The analysis of such distributions highlights discrepancies in the main definitions of PoS, leading us to prefer the one based on the utility function of the test. We illustrate our idea through an example and an application to clinical trials, which is a framework where PoS is commonly employed.

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Publication Details

Journal
Statistical Papers
Published
2024-09-19
DOI
https://doi.org/10.1007/s00362-024-01599-1
Citations
2
Primary Topic
Statistical Methods in Clinical Trials
Type
article
Field-Weighted Citation Impact
1.42
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article

The distribution of power-related random variables (and their use in clinical trials)

Stefania Gubbiotti, Francesco Mariani, Fulvio De Santis
2 citations
Statistical Papers
Statistical Methods in Clinical Trials
1.42
article

The distribution of power-related random variables (and their use in clinical trials)

Stefania Gubbiotti, Francesco Mariani, Fulvio De Santis
article en
2 citations

Abstract

Abstract In the hybrid Bayesian-frequentist approach to hypotheses tests, the power function, i.e. the probability of rejecting the null hypothesis, is a random variable and a pre-experimental evaluation of the study is commonly carried out through the so-called probability of success (PoS). PoS is usually defined as the expected value of the random power that is not necessarily a well-representative summary of the entire distribution. Here, we consider the main definitions of PoS and investigate the power related random variables that induce them. We provide general expressions for their cumulative distribution and probability density functions, as well as closed-form expressions when the test statistic is, at least asymptotically, normal. The analysis of such distributions highlights discrepancies in the main definitions of PoS, leading us to prefer the one based on the utility function of the test. We illustrate our idea through an example and an application to clinical trials, which is a framework where PoS is commonly employed.

Statistical PapersVol. 65(9)
Sapienza University of Rome (IT)
Openalex Percentile: Top 17%
Statistical Methods in Clinical Trials
1.42
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