Senior Clinical Pharmacologist Office of Clinical Evaluation, Office of Therapeutic Products, CBER/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
This presentation will provide an overview on the status and trend on the use of biomarker for cell and gene therapy products. We will summarize potential application of pharmacodynamic biomarkers and discuss clinical pharmacology principles in biomarker assessments of cell and gene therapy products. Illustrative cases in recent use of biomarkers in gene therapy products approvals will be provided. We also share some perspectives on the opportunities and challenges of utilizing biomarkers for characterizing mechanism of action, dose finding, providing confirmatory evidence of effectiveness, and establishing quantitative relationship between biomarkers and clinical outcomes for advanced medical products.
Learning Objectives:
Upon completion, the participants will understand and recognize the challenges associated with the use of biomarkers in rare diseases
Upon completion, the participants will be able to describe clinical pharmacology principles in biomarkers assessments of cell and gene therapies
Upon completion, the participants will learn about recent cases that illustrate use of biomarkers in cell and gene therapy products for rare diseases
