A promising biomarker adaptive Phase 2/3 design - Explained and expanded
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Updated: Jul 9, 2025

Predictive Immune Modeling of Solid Tumors
Published on: February 25, 2020
Cong Chen1, Linda Sun1, Xuekui Zhang2
1Biostatistics and Research Decision Sciences, Merck & Co., Inc., Rahway, NJ 07065, USA.
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.
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