Sample size re‐estimation in Phase 2 dose‐finding: Conditional power versus Bayesian predictive power. (23rd November 2022)

• Record Type: Journal Article
• Authors: Liu, Qingyang; Hu, Guanyu; Ye, Binqi; Wang, Susan; Wu, Yaoshi
• Is Part Of: Pharmaceutical statistics. Volume 22:Number 2(2023)
• Page Start: 349
• Page End: 364
• Publication Date: 2022-11-23
• Subjects: adaptive design -- clinical trial -- contrast test -- prior information -- sample size re‐estimation -- type I error; Pharmacy -- Statistical methods -- Periodicals; Pharmacy -- Statistics -- Periodicals; 615.10727
• DOI: 10.1002/pst.2275
• Languages: English
• ISSNs: 1539-1604

Abstract

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 (CP) and Bayesian predictive power (PP) 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.