Comparison of Bayesian methods for extrapolation of treatment effects: a large scale simulation study

Fuente: arXiv
Gespeichert in:
Bibliographische Detailangaben
Hauptverfasser: Fauvel, Tristan, Tanniou, Julien, Godbillot, Pascal, Génin, Marie, Amzal, Billy
Format: Preprint
Veröffentlicht: 2025
Schlagworte:
Online-Zugang:
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
_version_ 1866908670002987008
author Fauvel, Tristan
Tanniou, Julien
Godbillot, Pascal
Génin, Marie
Amzal, Billy
author_facet Fauvel, Tristan
Tanniou, Julien
Godbillot, Pascal
Génin, Marie
Amzal, Billy
contents Extrapolating treatment effects from related studies is a promising strategy for designing and analyzing clinical trials in situations where achieving an adequate sample size is challenging. Bayesian methods are well-suited for this purpose, as they enable the synthesis of prior information through the use of prior distributions. While the operating characteristics of Bayesian approaches for borrowing data from control arms have been extensively studied, methods that borrow treatment effects -- quantities derived from the comparison between two arms -- remain less well understood. In this paper, we present the findings of an extensive simulation study designed to address this gap. We evaluate the frequentist operating characteristics of these methods, including the probability of success, mean squared error, bias, precision, and credible interval coverage. Our results provide insights into the strengths and limitations of existing methods in the context of confirmatory trials. In particular, we show that the Conditional Power Prior and the Robust Mixture Prior perform better overall, while the test-then-pool variants and the p-value-based power prior display suboptimal performance.
format Preprint
id arxiv_https___arxiv_org_abs_2504_01949
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle Comparison of Bayesian methods for extrapolation of treatment effects: a large scale simulation study
Fauvel, Tristan
Tanniou, Julien
Godbillot, Pascal
Génin, Marie
Amzal, Billy
Methodology
Applications
Extrapolating treatment effects from related studies is a promising strategy for designing and analyzing clinical trials in situations where achieving an adequate sample size is challenging. Bayesian methods are well-suited for this purpose, as they enable the synthesis of prior information through the use of prior distributions. While the operating characteristics of Bayesian approaches for borrowing data from control arms have been extensively studied, methods that borrow treatment effects -- quantities derived from the comparison between two arms -- remain less well understood. In this paper, we present the findings of an extensive simulation study designed to address this gap. We evaluate the frequentist operating characteristics of these methods, including the probability of success, mean squared error, bias, precision, and credible interval coverage. Our results provide insights into the strengths and limitations of existing methods in the context of confirmatory trials. In particular, we show that the Conditional Power Prior and the Robust Mixture Prior perform better overall, while the test-then-pool variants and the p-value-based power prior display suboptimal performance.
title Comparison of Bayesian methods for extrapolation of treatment effects: a large scale simulation study
topic Methodology
Applications
url https://arxiv.org/abs/2504.01949