Stratified medicine in practice: review of predictive biomarkers in European Medicines Agency (EMA) indications

Objectives Stratified medicine has been defined as using a biomarker to match a patient to a cohort that has exhibited a differential response to a treatment. This is important where the proportion of patients benefiting from treatment is low and possible adverse events can be serious. To maximise patient benefit, valid predictive biomarkers need to be used. Several trial designs have been proposed to evaluate the use of predictive biomarkers in clinical practice including: enrichment, stratified and biomarker-based strategy design. Our aim was to review the EMA indications that include a predictive biomarker in order to investigate the type and strength of evidence considered sufficient for such decisions.