A semiparametric Bayesian approach to population finding with time‐to‐event and toxicity data in a randomized clinical trial

DOI10.1111/BIOM.13289MaRDI QIDQ6074511zbMATH OpenOpenAlexWikidataFDO

Authors Satoshi Morita, P. Müller, Hiroyasu Abe

Publication date 12 October 2023

Published in Biometrics (Search for Journal in Brave)

Full work available at URL https://arxiv.org/abs/1910.12174

zbMATH Keywords

Bayesian additive regression treesBayesian decision problemefficacy-toxicity trade-offnonparametric Bayesianpopulation findingPólya tree prior

Mathematics Subject Classification ID

Applications of statistics to biology and medical sciences; meta analysis (62P10)

Abstract: A utility-based Bayesian population finding (BaPoFi) method was proposed by Morita and M"uller (2017, Biometrics, 1355-1365) to analyze data from a randomized clinical trial with the aim of identifying good predictive baseline covariates for optimizing the target population for a future study. The approach casts the population finding process as a formal decision problem together with a flexible probability model using a random forest to define a regression mean function. BaPoFi is constructed to handle a single continuous or binary outcome variable. In this paper, we develop BaPoFi-TTE as an extension of the earlier approach for clinically important cases of time-to-event (TTE) data with censoring, and also accounting for a toxicity outcome. We model the association of TTE data with baseline covariates using a semi-parametric failure time model with a P'olya tree prior for an unknown error term and a random forest for a flexible regression mean function. We define a utility function that addresses a trade-off between efficacy and toxicity as one of the important clinical considerations for population finding. We examine the operating characteristics of the proposed method in extensive simulation studies. For illustration, we apply the proposed method to data from a randomized oncology clinical trial. Concerns in a preliminary analysis of the same data based on a parametric model motivated the proposed more general approach.

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(3)

• Bayesian population finding with biomarkers in a randomized clinical trial
• Biomarker-based Bayesian randomized clinical trial design for identifying a target population
• Exploring sensitive biomarkers associated with short-term responses and long-term outcomes using Bayesian additive regression trees

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