A Review of Bayesian Perspectives on Sample Size Derivation for Confirmatory Trials.
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Authors
Kunzmann, Kevin
Grayling, Michael J
Lee, Kim May
Robertson, David S
Rufibach, Kaspar
Wason, James MS
Publication Date
2021Journal Title
Am Stat
ISSN
0003-1305
Publisher
Informa UK Limited
Volume
75
Issue
4
Pages
424-432
Language
eng
Type
Article
This Version
VoR
Metadata
Show full item recordCitation
Kunzmann, K., Grayling, M. J., Lee, K. M., Robertson, D. S., Rufibach, K., & Wason, J. M. (2021). A Review of Bayesian Perspectives on Sample Size Derivation for Confirmatory Trials.. Am Stat, 75 (4), 424-432. https://doi.org/10.1080/00031305.2021.1901782
Description
Funder: Biometrika Trust
Abstract
Sample size derivation is a crucial element of planning any confirmatory trial. The required sample size is typically derived based on constraints on the maximal acceptable Type I error rate and minimal desired power. Power depends on the unknown true effect and tends to be calculated either for the smallest relevant effect or a likely point alternative. The former might be problematic if the minimal relevant effect is close to the null, thus requiring an excessively large sample size, while the latter is dubious since it does not account for the a priori uncertainty about the likely alternative effect. A Bayesian perspective on sample size derivation for a frequentist trial can reconcile arguments about the relative a priori plausibility of alternative effects with ideas based on the relevance of effect sizes. Many suggestions as to how such "hybrid" approaches could be implemented in practice have been put forward. However, key quantities are often defined in subtly different ways in the literature. Starting from the traditional entirely frequentist approach to sample size derivation, we derive consistent definitions for the most commonly used hybrid quantities and highlight connections, before discussing and demonstrating their use in sample size derivation for clinical trials.
Keywords
Power, Assurance, Probability Of Success, Expected Power, Sample Size Derivation
Sponsorship
Medical Research Council (MC_UU_00002/14)
Identifiers
34992303, PMC7612172
External DOI: https://doi.org/10.1080/00031305.2021.1901782
This record's URL: https://www.repository.cam.ac.uk/handle/1810/333777
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