Prologue: Biomarker Strategies in Therapeutics Development for Rare Diseases

On Demand

Location:Online Only

Prologue Speaker(s)

Yow-Ming Wang, PhD

Associate Director for Biosimilars and Therapeutic Biologics
US Food and Drug Administration
Silver Spring, Maryland

This prologue presentation will provide the reader with a regulatory context for rare disease drug development and using examples of recent FDA approvals to illustrate the application of innovative approaches with biomarker evaluations. Topics covered in the presentation are
(1) The goal of therapeutic drug development & regulatory expectations,
(2) FDA’s efforts on addressing challenges related to rare disease drug development,
(3) Biomarkers – definitions and utilities of response biomarkers,
(4) Examples of FDA approvals illustrating the use of innovative approaches including biomarkers, and
(5) Biomarker strategy includes bioanalytical considerations.

To view all Prologues, visit our library here and click "Enroll".

Learning Objectives:

To discuss the regulatory expectations and the FDA's efforts in facilitating and advancing the development of therapeutics for rare diseases.
To leverage the resources on the FDA's websites in developing innovative approaches for their rare disease therapeutic development program.
To learn from past approvals of rare disease therapeutics and explore developing confirmatory evidence to support their clinical program, including the use of biomarkers.
To implement biomarker strategy with thoughtful consideration of bioanalytical method validation when the biomarker data are planned for supporting regulatory decision-making.