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Flexible Conditional Borrowing Approaches for Leveraging Historical Data in the Bayesian Design of Superiority Trials

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Abstract

In this paper, we consider the Bayesian design of a randomized, double-blind, placebo-controlled superiority clinical trial. To leverage multiple historical datasets to augment the placebo-controlled arm, we develop three conditional borrowing approaches built upon the borrowing-by-parts prior, the hierarchical prior, and the robust mixture prior. The operating characteristics of the conditional borrowing approaches are examined. Extensive simulation studies are carried out to empirically demonstrate the superiority of the conditional borrowing approaches over the unconditional borrowing or no-borrowing approaches in terms of controlling type I error, maintaining good power, having a large “sweet-spot” region, minimizing bias, and reducing the mean-squared error of the posterior estimate of the mean parameter of the placebo-controlled arm. Computational algorithms are also developed for calculating the Bayesian type I error and power as well as the corresponding simulation errors.

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Acknowledgements

We would like to thank the Editors-in-Chief, the Guest Editors, and two reviewers for their comments and suggestions, which have led to a much improved version of this paper. Ms. Yuan and Dr. Chen’s research was partially supported by REGENXBIO Inc., and Dr. Chen’s research was also partially supported by NIH Grants #GM70335 and #P01CA142538.

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Authors and Affiliations

  1. Department of Statistics, University of Connecticut at Storrs, Storrs, CT, USA

    Wenlin Yuan & Ming-Hui Chen

  2. REGENXBIO Inc, 9600 Blackwell Road, Suite 210, Rockville, MD, USA

    John Zhong

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  1. Wenlin Yuan
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  2. Ming-Hui Chen
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  3. John Zhong
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Correspondence to Ming-Hui Chen.

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Yuan, W., Chen, MH. & Zhong, J. Flexible Conditional Borrowing Approaches for Leveraging Historical Data in the Bayesian Design of Superiority Trials. Stat Biosci 14, 197–215 (2022). https://doi.org/10.1007/s12561-021-09321-7

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  • DOI: https://doi.org/10.1007/s12561-021-09321-7

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