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Updated: Jan 11, 2026

Selecting Multiple Biomarker Subsets with Similarly Effective Binary Classification Performances
Published on: October 11, 2018
Optimality Considerations in Two-Stage Biomarker-Stratified Trials With Adaptive Enrichment
1Department of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
None:
We consider biomarker-stratified trials that test the treatment effect in a targeted patient group, the biomarker positive subgroup, and in the overall population. If the biomarker negative subgroup is stopped for futility during the interim analysis the number of participants assigned can be increased. Optimal two-stage designs are obtained using several optimality criteria. Recommendations are given on the best interim analysis timing, futility boundaries, α allocation between the two hypotheses and stage 2 sample size for the biomarker positive subgroup in case its complement, the biomarker negative subgroup, is stopped for futility in the interim analysis. This work is motivated by PrecISE, a two-stage biomarker-stratified clinical trial in patients with severe asthma.
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