Statistics in Oncology Series
Biomarker Discovery and Validation: Statistical ConsiderationsBiomarkers have various applications including disease detection, diagnosis, prognosis, prediction of response to intervention, and disease monitoring. In this era of precision medicine, having validated biomarkers to inform clinical decision making is more important than ever. In this article, we discuss best the practices and potential issues in biomarker discovery and validation. We encourage team science partnerships to bring cutting-edge discovery from bench to bedside, leading to improved patient care and outcomes.
All-Comers versus Enrichment Design Strategy in Phase II TrialsDesigns for biomarker validation have been proposed and used in the phase III oncology clinical trial setting. Broadly, these designs follow either an enrichment (i.e., targeted) strategy or an all-comers (i.e., unselected) strategy. An enrichment design screens patients for the presence or absence of a marker or a panel of markers and then only includes patients who either have or do not have a certain marker characteristic or profile. In contrast, all patients meeting the eligibility criteria (regardless of a particular biomarker status) are entered into an all-comers design.