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Case-only analysis in small studies of predictive biomarkers

M Hauptmann1, V H Nguyen2,3, L Sollfrank2

  • 1Brandenburg Medical School Theodor Fontane, Institute of Biostatistics and Registry Research, Fehrbelliner Straße 39, 16816, Neuruppin, Germany. michael.hauptmann@mhb-fontane.de.

Scientific Reports
|April 16, 2025
PubMed
Summary

Case-only analysis for biomarkers in cancer treatment selection is generally inferior to full cohort analysis. However, it shows promise for cost savings in specific scenarios, like rare events and independent treatment assignment.

Keywords:
Biomarker-treatment interactionCase-only analysisFirth’s penalized maximum likelihoodTreatment heterogeneity

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