ABSTRACT
The prognostic biomarker is used to identify the likelihood of a clinical event, disease recurrence, or progression in patients who have the disease or medical condition of interest. These biomarkers are always identified from observational data and used to identify patients more likely to have a particular outcome. This chapter focuses on trial designs for biomarker validation, including in the setting of the early-phase trials and confirmatory trials. The biomarker-stratified design aims to randomly assign patients into different intervention groups either based on the biomarker status. This design includes patients with both biomarker positive and biomarker negative who are treated with the same regimen, resulting in a significant overlap (driven by the prevalence of the marker) in the number of patients receiving the same treatment regimen in both arms. Co-development imposes the necessity to generate specific guidelines describing analytical test validation, clinical test validation and clinical utility.
