Regulatory Science Objectives and Biomarker Qualification Through Public-Private Partnerships Are Critical to Delivering Innovative Treatments for CNS Diseases

Abstract Central nervous system (CNS) diseases represent one of the most challenging therapeutic areas for successful drug approvals. Developing biomarkers as drug development tools (DDTs) that catalyze the path to innovative treatments can aid in reducing the risk and improving the chances of approvals. Biomarker development, validation, and regulatory endorsement are a similarly rigorous process. Regulatory endorsement of biomarkers is a key goal for all stakeholders, and several paths exist with the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). This chapter highlights the regulatory paths and considerations for advancing biomarker use in clinical development. Advancing biomarkers for CNS diseases is critical for advancing innovative therapeutics. Public-private partnerships (PPPs) provide the infrastructure for bringing together data and all relevant stakeholders in a precompetitive platform aimed at sharing costs and risks.