Literature DB >> 29717945

Bayesian sample size re-estimation using power priors.

T B Brakenhoff1, Kcb Roes1, S Nikolakopoulos1.   

Abstract

The sample size of a randomized controlled trial is typically chosen in order for frequentist operational characteristics to be retained. For normally distributed outcomes, an assumption for the variance needs to be made which is usually based on limited prior information. Especially in the case of small populations, the prior information might consist of only one small pilot study. A Bayesian approach formalizes the aggregation of prior information on the variance with newly collected data. The uncertainty surrounding prior estimates can be appropriately modelled by means of prior distributions. Furthermore, within the Bayesian paradigm, quantities such as the probability of a conclusive trial are directly calculated. However, if the postulated prior is not in accordance with the true variance, such calculations are not trustworthy. In this work we adapt previously suggested methodology to facilitate sample size re-estimation. In addition, we suggest the employment of power priors in order for operational characteristics to be controlled.

Keywords:  Bayesian; Sample size; borrowing; monitoring; power prior; randomized controlled trial; re-estimation; variance

Year:  2018        PMID: 29717945     DOI: 10.1177/0962280218772315

Source DB:  PubMed          Journal:  Stat Methods Med Res        ISSN: 0962-2802            Impact factor:   3.021


  3 in total

Review 1.  Bayesian Approaches for Confirmatory Trials in Rare Diseases: Opportunities and Challenges.

Authors:  Moreno Ursino; Nigel Stallard
Journal:  Int J Environ Res Public Health       Date:  2021-01-24       Impact factor: 3.390

2.  Applicability and added value of novel methods to improve drug development in rare diseases.

Authors:  Marian Mitroiu; Katrien Oude Rengerink; Caridad Pontes; Aranzazu Sancho; Roser Vives; Stella Pesiou; Juan Manuel Fontanet; Ferran Torres; Stavros Nikolakopoulos; Konstantinos Pateras; Gerd Rosenkranz; Martin Posch; Susanne Urach; Robin Ristl; Armin Koch; Spineli Loukia; Johanna H van der Lee; Kit C B Roes
Journal:  Orphanet J Rare Dis       Date:  2018-11-12       Impact factor: 4.123

3.  Designing Clinical Trials in Wilson's Disease.

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Journal:  Hepatology       Date:  2021-10-05       Impact factor: 17.298

  3 in total

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