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Using Cholesky Decomposition to Explore Individual Differences in Longitudinal Relations between Reading Skills
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Bayesian borrowing for basket trials with longitudinal outcomes.

Lou E Whitehead1, Oliver Sailer2, Miles D Witham3

  • 1Biostatistics Research Group, Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK.

Statistics in Medicine
|April 30, 2023
PubMed
Summary
This summary is machine-generated.

Bayesian borrowing methods enhance clinical trial power for aging-related diseases by sharing information across patient subgroups in basket trials. These methods improve treatment effect detection and precision, especially for longitudinal outcomes.

Keywords:
Bayesian hierarchical modelingBayesian statisticsbasket trialsborrowing informationlongitudinal

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Area of Science:

  • Biostatistics
  • Clinical Trial Design
  • Aging Research

Background:

  • Basket trials enable single intervention investigation across multiple patient subgroups.
  • These trials offer advantages like reduced sample sizes and increased efficiency.
  • Existing methods for basket trials are limited for chronic aging-related diseases with longitudinal outcomes.

Purpose of the Study:

  • To extend Bayesian borrowing methods for basket designs with continuous longitudinal endpoints.
  • To facilitate the application of basket trials in aging-related disease research.
  • To evaluate the performance of information-sharing methods against standalone analyses.

Main Methods:

  • Extension of three Bayesian borrowing methods for basket trials.
  • Application to a real-world dataset and a simulation study.
  • Comparison with independent analysis of each basket without borrowing.

Main Results:

  • Information-sharing methods improve power and precision for detecting treatment effects.
  • In heterogeneous scenarios, a trade-off exists between increased power and Type I error risk.
  • Proposed methods demonstrate utility for basket trials with longitudinal outcomes.

Conclusions:

  • Bayesian borrowing methods enhance power and precision in basket trials for aging-related diseases.
  • Careful method selection is crucial, considering trial priorities and expected treatment effect distributions.
  • These methods support the use of basket designs for complex chronic diseases.