Sample size re-estimation in Phase 2 Dose-Finding: Conditional power vs. Bayesian predictive power

2020/12/29 by Qingyang Liu, Liu, Qingyang, Guanyu Hu +7
Decision Sciences · Mathematics · #Applications (stat.AP) #FOS: Computer and information sciences #Methodology (stat.ME) #Optimal Experimental Design Methods #Statistical Methods in Clinical Trials

paper · pdf · doi:10.48550/arxiv.2012.14589

openalex publication_date 2020/12/29 · openalex created_date 2022/11/16 · openalex updated_date 2026/07/28

Abstract

Unblinded sample size re-estimation (SSR) is often planned in a clinical trial when there is large uncertainty about the true treatment effect. For Proof-of Concept (PoC) in a Phase II dose finding study, contrast test can be adopted to leverage information from all treatment groups. In this article, we propose two-stage SSR designs using frequentist conditional power and Bayesian posterior predictive power for both single and multiple contrast tests. The Bayesian SSR can be implemented under a wide range of prior settings to incorporate different prior knowledge. Taking the adaptivity into account, all type I errors of final analysis in this paper are rigorously protected. Simulation studies are carried out to demonstrate the advantages of unblinded SSR in multi-arm trials.

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