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

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...

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Bibliographic Details
Main Authors Liu, Qingyang, Hu, Guanyu, Ye, Binqi, Wang, Susan, Wu, Yaoshi
Format Journal Article
LanguageEnglish
Published 28.12.2020
Subjects
Statistics - Applications
Statistics - Methodology
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Summary: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.
DOI:10.48550/arxiv.2012.14589