Salsbury, J.A., Oakley, J.E. orcid.org/0000-0002-9860-4093, Julious, S.A. et al. (1 more author) (2024) Assurance methods for designing a clinical trial with a delayed treatment effect. Statistics in Medicine, 43 (19). pp. 3595-3612. ISSN 0277-6715
Abstract
An assurance calculation is a Bayesian alternative to a power calculation. One may be performed to aid the planning of a clinical trial, specifically setting the sample size or to support decisions about whether or not to perform a study. Immuno-oncology is a rapidly evolving area in the development of anticancer drugs. A common phenomenon that arises in trials of such drugs is one of delayed treatment effects, that is, there is a delay in the separation of the survival curves. To calculate assurance for a trial in which a delayed treatment effect is likely to be present, uncertainty about key parameters needs to be considered. If uncertainty is not considered, the number of patients recruited may not be enough to ensure we have adequate statistical power to detect a clinically relevant treatment effect and the risk of an unsuccessful trial is increased. We present a new elicitation technique for when a delayed treatment effect is likely and show how to compute assurance using these elicited prior distributions. We provide an example to illustrate how this can be used in practice and develop open-source software to implement our methods. Our methodology has the potential to improve the success rate and efficiency of Phase III trials in immuno-oncology and for other treatments where a delayed treatment effect is expected to occur.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2024 The Author(s). Statistics in Medicine published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits use, distribution and reproduction in any medium, provided the original work is properly cited. |
Keywords: | assurance; expert judgement; prior elicitation; delayed treatment effects; probability of success |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Science (Sheffield) > School of Mathematics and Statistics (Sheffield) |
Funding Information: | Funder Grant number Engineering and Physical Sciences Research Council 2610753 |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 07 Jun 2024 10:41 |
Last Modified: | 20 Nov 2024 16:20 |
Status: | Published |
Publisher: | Wiley |
Refereed: | Yes |
Identification Number: | 10.1002/sim.10136 |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:212988 |