Designs 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. The strength of the preliminary evidence, the prevalence of the marker, the reproducibility and validity of the assay, and the feasibility of real-time marker assessment play a major role in the choice of the design. In this report, we discuss the parameters under which the enrichment or an all-comers design strategy would be appropriate for phase II trials.
- Phase II
ASJC Scopus subject areas
- Pulmonary and Respiratory Medicine