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Sample size calculation in randomised phase II selection trials using a margin of practical equivalence

Dehbi, H-M; Hackshaw, A; (2020) Sample size calculation in randomised phase II selection trials using a margin of practical equivalence. Trials , 21 , Article 301. 10.1186/s13063-020-04248-8. Green open access

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Abstract

BACKGROUND: In rare cancers or subtypes of common cancers, a comparison of multiple promising treatments may be required. The selected treatment can then be assessed against the standard of care (if it exists) or used as a backbone for combinations with new, possibly targeted, agents. There could be different experimental therapies or different doses of the same therapy, and either can be done in combination with standard treatments. A 'pick-the-winner' design is often used, which focuses on efficacy to select the most promising treatment. However, a treatment with a slightly lower efficacy compared to another treatment may actually be preferred if it has a better toxicity or quality of life profile, is easier to administer, or cheaper. METHODS: By pre-defining a margin of practical equivalence in order to calculate the sample size, a more flexible assessment can be made of whether the treatments have very different effects or are sufficiently close so that other factors can be used to choose between them. Using exact binomial probabilities, we calculated the sample size for two- and three-arm randomised selection trials including a margin of practical equivalence with a variety of input parameters. RESULTS: We explain conceptually the margin of practical equivalence in this paper, and provide a free user-friendly web application to calculate the required sample size for a variety of input parameters. CONCLUSION: The web application should help promote the randomised selection design with a margin of practical equivalence, which provides greater flexibility than the 'pick-the-winner' approach in assessing the results of selection trials.

Type: Article
Title: Sample size calculation in randomised phase II selection trials using a margin of practical equivalence
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1186/s13063-020-04248-8
Publisher version: https://doi.org/10.1186/s13063-020-04248-8
Language: English
Additional information: This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
Keywords: Randomised trial, Rare cancers, Selection design
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Cancer Institute > CRUK Cancer Trials Centre
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology > Comprehensive CTU at UCL
URI: https://discovery.ucl.ac.uk/id/eprint/10094918
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