Estimating design operating characteristics in Bayesian adaptive clinical trials

DOI10.1002/CJS.11699MaRDI QIDQ5094306zbMATH OpenOpenAlexFDO

Authors Shirin Golchi

Publication date 2 August 2022

Published in The Canadian Journal of Statistics (Search for Journal in Brave)

Full work available at URL https://arxiv.org/abs/2105.03022

zbMATH Keywords

Bayesian test statisticconstrained designCOVID-19ordinal-scale outcomeproportional-odds modelsampling distributiontrial simulation

Mathematics Subject Classification ID

Bayesian problems; characterization of Bayes procedures (62C10) Parametric hypothesis testing (62F03) Bayesian inference (62F15) Density estimation (62G07) Optimal stopping in statistics (62L15) Inference from spatial processes (62M30) Applications of statistics to biology and medical sciences; meta analysis (62P10)

Abstract: Bayesian adaptive designs have gained popularity in all phases of clinical trials with numerous new developments in the past few decades. During the COVID-19 pandemic, the need to establish evidence for the effectiveness of vaccines, therapeutic treatments and policies that could resolve or control the crisis emphasized the advantages offered by efficient and flexible clinical trial designs. In many COVID-19 clinical trials, due to the high level of uncertainty, Bayesian adaptive designs were considered advantageous. Designing Bayesian adaptive trials, however, requires extensive simulation studies that are generally considered challenging, particularly in time-sensitive settings such as a pandemic. In this article, we propose a set of methods for efficient estimation and uncertainty quantification for design operating characteristics of Bayesian adaptive trials. Specifically, we model the sampling distribution of Bayesian probability statements that are commonly used as the basis of decision making. To showcase the implementation and performance of the proposed approach, we use a clinical trial design with an ordinal disease-progression scale endpoint that was popular among COVID-19 trial. However, the proposed methodology may be applied generally in clinical trial context where design operating characteristics cannot be obtained analytically.

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Cited in

(8)

• Predictive Hierarchic Modeling of Operational Characteristics in Clinical Trials
• scientific article; zbMATH DE number 5898591 (Why is no real title available?)
• Parameter estimation following an adaptive treatment selection trial design
• Generalized Likelihood Ratio Statistics and Uncertainty Adjustments in Efficient Adaptive Design of Clinical Trials
• Bayesian Response-Adaptive Designs for Basket Trials
• Comment: advancing clinical trials with novel designs and implementations
• An Economical Approach to Design Posterior Analyses
• Estimating the sampling distribution of posterior decision summaries in Bayesian clinical trials

Describes a project that uses

Uses Software

• bayesCPM

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