Volume 22, Issue 3 pp. 531-546
MAIN PAPER

Bayesian hierarchical models for adaptive basket trial designs

Chian Chen

Chian Chen

Institute of Population Health Sciences, National Health Research Institutes, Miaoli County, Taiwan

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Chin-Fu Hsiao

Corresponding Author

Chin-Fu Hsiao

Institute of Population Health Sciences, National Health Research Institutes, Miaoli County, Taiwan

Correspondence

Chin-Fu Hsiao, Institute of Population Health Sciences, National Health Research Institutes, 35 Keyan Road, Zhunan, Miaoli County 35053, Taiwan.

Email: [email protected]

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First published: 10 January 2023

Abstract

Basket trials evaluate a single drug targeting a single genetic variant in multiple cancer cohorts. Empirical findings suggest that treatment efficacy across baskets may be heterogeneous. Most modern basket trial designs use Bayesian methods. These methods require the prior specification of at least one parameter that permits information sharing across baskets. In this study, we provide recommendations for selecting a prior for scale parameters for adaptive basket trials by using Bayesian hierarchical modeling. Heterogeneity among baskets attracts much attention in basket trial research, and substantial heterogeneity challenges the basic assumption of exchangeability of Bayesian hierarchical approach. Thus, we also allowed each stratum-specific parameter to be exchangeable or nonexchangeable with similar strata by using data observed in an interim analysis. Through a simulation study, we evaluated the overall performance of our design based on statistical power and type I error rates. Our research contributes to the understanding of the properties of Bayesian basket trial designs.

CONFLICT OF INTEREST

The authors declare no conflict of interest.

DATA AVAILABILITY STATEMENT

All data generated or used during the study appear in the submitted article (or in reference).

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