Regulatory Perspectives on the Use of Biomarkers and Personalized Medicine in CNS Drug Development: The FDA Viewpoint

Abstract

Abstract Drug development for central nervous system (CNS) disorders is challenging and has faced a mixed bag of successes. Failures to identify safe and effective therapies for conditions such as Alzheimer s disease and Parkinson s disease have been attributed to lack of good preclinical disease models, absences of link between biomarkers, and clinical end points and adequate heterogeneity in disease progression. Understanding reasons for the disease heterogeneity and identifying early markers for treatment effect have been recognized as important factors for successful drug development for CNS disorders. Recent scientific advances in genetic sequencing and imaging technologies offer promising ways to accelerate the development of therapies. Toward achieving this goal, several FDA regulatory initiatives, such as biomarker qualification program (BQP), Critical Path Innovations Meeting (CPIM), and expedited drug approval pathways, could potentially lead to successful and more targeted CNS therapies in the future.