Despite the existence of more than 7,000 rare diseases, only a small number of diseases have therapeutics currently available. Developing therapies for rare neurodegenerative diseases is particularly challenging due to limited understanding, low prevalence, disease heterogeneity, high risk, and limited commercial incentives. This presentation will demonstrate how biomarkers played a critical role in the development and recent approval of drug development programs in the rare neurodegenerative diseases. It will showcase examples of biomarkers used in different stages of drug development, including proof-of-concept, dose-selection, patient population selection, and supporting changes in route of administration or dosing frequency. The presentation will also discuss the role of biomarkers in regulatory decision-making, including their use as surrogate endpoints for accelerated approval or traditional approval. The overall goal of this presentation is to emphasize the importance of biomarkers in drug development and regulatory decision-making for the success of new therapies in neurology.
Learning Objectives:
Describe the different stages of drug development where biomarkers can be used, including proof-of-concept, dose-selection, patient population selection, and supporting changes in route of administration or dosing frequency.
Explain how biomarkers can be utilized as surrogate endpoints for accelerated approval or traditional approval in regulatory decision-making and describe the FDA's review process of biomarkers.
Analyze recently approved neurology drugs and their biomarkers to understand the vital role of biomarkers in drug development and regulatory decision-making through case examples.
