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Updated: May 23, 2026
07:35
Selecting Multiple Biomarker Subsets with Similarly Effective Binary Classification Performances
Published on: October 11, 2018
Biomarkers, subgroup evaluation, and clinical trial design
Stuart G Baker1, Barnett S Kramer, Daniel J Sargent
1Division of Cancer Prevention, National Cancer Institute, Bethesda, Maryland 20892, USA. sb16i@nih.gov
Discovery Medicine
|April 3, 2012
Summary
Identifying patient subgroups for targeted treatments requires specialized clinical trial designs. The biomarker-analysis and adaptive signature designs help pinpoint which patients benefit most from therapies, optimizing treatment efficacy.
Area of Science:
- Biomarker discovery
- Clinical trial methodology
- Translational medicine
Background:
- Advances in genetic and clinical biomarkers offer potential for personalized medicine.
- Identifying patient subgroups who benefit from specific treatments is crucial for therapeutic success.
Purpose of the Study:
- To outline specialized clinical trial designs for identifying and evaluating patient subgroups based on biomarkers.
- To present the biomarker-analysis and adaptive signature designs for optimizing treatment benefit assessment.
Main Methods:
- The biomarker-analysis design involves randomized trials with subgroup analysis based on biomarker cutpoints.
- The adaptive signature design uses training and test samples to formulate subgroup rules and estimate treatment benefit.
- The sliding-window subgroup plot can extend the adaptive signature design.
Main Results:
- The biomarker-analysis design is optimal for single continuous biomarkers.
- The adaptive signature design is recommended for identifying subgroups using multiple markers.
- Extensions like the sliding-window subgroup plot enhance subgroup evaluation.
Conclusions:
- Specialized clinical trial designs are essential to leverage biomarker potential for personalized medicine.
- The biomarker-analysis and adaptive signature designs provide robust frameworks for subgroup identification and treatment evaluation.
- These designs facilitate more precise and effective therapeutic strategies.
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