Augmented binary method for basket trials (ABBA)

In several clinical areas, traditional clinical trials often use a responder outcome, a composite endpoint that involves dichotomising a continuous measure. An augmented binary method that improves power while retaining the original responder endpoint has previously been proposed. The method leverages information from the undichotomised component to improve power. We extend this method for basket trials, which are gaining popularity in many clinical areas. For clinical areas where response outcomes are used, we propose the new augmented binary method for basket trials that enhances efficiency by borrowing information on the treatment effect between subtrials. The method is developed within a latent variable framework using a Bayesian hierarchical modelling approach. We investigate the properties of the proposed methodology by analysing point estimates and high-density intervals in various simulation scenarios, comparing them to the standard analysis for basket trials that assumes binary outcomes. Our method results in a reduction of 95% high-density interval of the posterior distribution of the log odds ratio and an increase in power when the treatment effect is consistent across subtrials. We illustrate our approach using real data from two clinical trials in rheumatology.

• Publication year

  2024

• Venue

  Statistical Methods in Medical Research

• Publication date

  2024-08-16

• Fields of study

  Medicine, Mathematics

• Identifiers
  DOI 10.1177/09622802251403365arXiv 2408.08636PMID 41348568PMCID 12824627
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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