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Lookup NU author(s): Dr Haiyan ZhengORCiD, Professor James WasonORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
Basket trials have emerged as a new class of efficient approaches in oncology to evaluate a new treatment in several patient subgroups simultaneously. In this paper, we extend the key ideas to disease areas outside of oncology, developing a robust Bayesian methodology for randomised, placebo-controlled basket trials with a continuous endpoint to enable borrowing of information across subtrials with similar treatment effects. After adjusting for covariates, information from a complementary subtrial can be represented into a commensurate prior for the parameter that underpins the subtrial under consideration. We propose using distributional discrepancy to characterise the commensurability between subtrials for appropriate borrowing of information through a spike-and-slab prior, which is placed on the prior precision factor. When the basket trial has at least three subtrials, commensurate priors for point-to-point borrowing are combined into a marginal predictive prior, according to the weights transformed from the pairwise discrepancy measures. In this way, only information from subtrial(s) with the most commensurate treatment effect is leveraged. The marginal predictive prior is updated to a robust posterior by the contemporary subtrial data to inform decision making. Operating characteristics of the proposed methodology are evaluated through simulations motivated by a real basket trial in chronic diseases. The proposed methodology has advantages compared to other selected Bayesian analysis models, for (i) identifying the most commensurate source of information, and (ii) gauging the degree of borrowing from specific subtrials. Numerical results also suggest that our methodology can improve the precision of estimates and, potentially, the statistical power for hypothesis testing.
Author(s): Zheng H, Wason JMS
Publication type: Article
Publication status: Published
Journal: Biostatistics
Year: 2022
Volume: 23
Issue: 1
Pages: 120-135
Print publication date: 01/01/2022
Online publication date: 07/05/2020
Acceptance date: 25/03/2020
Date deposited: 27/05/2020
ISSN (print): 1465-4644
ISSN (electronic): 1468-4357
Publisher: Oxford University Press
URL: https://doi.org/10.1093/biostatistics/kxaa019
DOI: 10.1093/biostatistics/kxaa019
Notes: arXiv preprint arXiv:1908.05091
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