A promising biomarker adaptive Phase 2/3 design - Explained and expanded

Cong Chen; Linda Sun; Xuekui Zhang

doi:10.1016/j.conctc.2023.101229

A promising biomarker adaptive Phase 2/3 design - Explained and expanded

Contemp Clin Trials Commun. 2023 Nov 10:36:101229. doi: 10.1016/j.conctc.2023.101229. eCollection 2023 Dec.

Authors

Cong Chen¹, Linda Sun¹, Xuekui Zhang²

Affiliations

¹ Biostatistics and Research Decision Sciences, Merck & Co., Inc., Rahway, NJ 07065, USA.
² Department of Mathematics and Statistics, University of Victoria, Canada.

Abstract

This short communication concerns a biomarker adaptive Phase 2/3 design for new oncology drugs with an uncertain biomarker effect. Depending on the outcome of an interim analysis for adaptive decision, a Phase 2 study that starts in a biomarker enriched subpopulation may continue to the end without expansion to Phase 3, expand to Phase 3 in the same population or expand to Phase 3 in a broader population. Each path can enjoy full alpha for hypothesis testing without inflating the overall Type I error.