ABSTRACT
This chapter provides an overview of classical and adaptive designs to consider if biomarkers are to be incorporated into clinical trials. Biomarker adaptive clinical trials are useful in studying rare diseases, particularly considering that the availability of patients with rare diseases is quite limited. The biomarker-adaptive threshold design combines the test of overall treatment effect with the establishment and validation of a cut point for a pre-specified biomarker which identifies a biomarker-based subgroup believed to be most sensitive to the experimental treatment. The adaptive signature designs a design proposed to select the subgroup using a large number of potential biomarkers. Precision medicine can potentially bring the following additional benefits to patients beyond the “traditional” approach to prescribing medicines: ability to make more informed medical decisions, higher probability of desired outcomes, minimize the risk of side effects, focus on prevention of disease rather than reaction to it, and reduced healthcare costs.
