Since the passage of 21st Century Cures, FDA collaboration with the rare disease patient and scientific community has never been greater. The creation of a Rare Disease Innovation Hub also holds promise, but barriers remain to scientific dialogue and the agency in an ever-evolving landscape. The Externally Led-Scientific Focused Drug Development meeting (EL-SFDD) seeks to bridge the gap between rare disease expertise and regulatory expertise. Legislation has been introduced in Congress to create these quarterly meetings to provide an opportunity for enhanced to discuss the challenges impacting the development of rare disease treatments, identify scientific opportunities, discuss novel clinical trial designs, and align on endpoints to address unmet medical needs for rare disease patients Heard directly from regulatory, scientific and patient organizations who have lived the benefits of this approach and learn how their experiences could shape the future of rare disease.
Moderator:
- Chris Porter, Vice President, Travere Therapeutics
Speakers:
- James C. Shehan, Senior Counsel; Chair, FDA Regulatory, Lowenstein Sandler LLP
- Barbara Gillespie, MD, FASN, Therapeutic Head of Nephrology, Fortrea; Adjunct Professor, University of North Carolina, Division of Nephrology and Hypertension
- Jamie Sullivan, MPH, Vice President of Policy, EveryLife Foundation for Rare Diseases
- Jackie Weinrich, Health Policy Advisor, Rep. Doris Matsui
Time: 11 a.m.-12 p.m. ET
Location: Boston Convention & Exhibition Center, 415 Summer St, Boston, MA 02210
*This event is open to the public but requires a registration fee.
