Sample size re-estimation in Phase 2 dose-finding: Conditional power versus Bayesian predictive power

Pharm Stat. 2023 Mar;22(2):349-364. doi: 10.1002/pst.2275. Epub 2022 Nov 23.

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.

Keywords: adaptive design; clinical trial; contrast test; prior information; sample size re-estimation; type I error.

Publication types

  • Clinical Trial, Phase II
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Bayes Theorem
  • Computer Simulation
  • Humans
  • Research Design*
  • Sample Size
  • Uncertainty