Staff Fellow Office of Clinical Pharmacology, Office of Translational Sciences, CDER/FDA Silver Spring, Maryland
This 30-minute presentation is one of the four presentations in a two-hour symposium, which is proposed by Drs. Yow-Ming Wang and Xiaofei Wang. The title of the two-hour symposium is Biomarker Strategies in Therapeutics Development for Rare Diseases.
Biomarkers play a pivotal role in supporting drug approval for rare diseases. They offer crucial insights into the efficacy and safety of experimental therapies by serving as measurable indicators of treatment response. Their utilization can significantly enhance clinical trial efficiency, particularly in the context of rare diseases, where patient populations are limited, and traditional trial methodologies may be impractical. This presentation will showcase two case studies, each focusing on a different rare neurological disease: Amyotrophic Lateral Sclerosis and Duchenne Muscle Dystrophy. In both case studies, biomarkers have served as central elements in identifying treatment effects within a limited, heterogeneous disease population, thereby influencing regulatory decision-making.
Learning Objectives:
Upon completion, participant will understand how biomarkers contribute to the Accelerated Approval for treatments targeting rare neurological diseases.
Upon completion, participant will gain insights into the practical applications of biomarkers in developing treatments for rare neurological disorders.
Upon completion, participant will learn the pivotal role of biomarkers supporting drug approval for rare diseases
