Orphans In Jeopardy: Threats to the Development of Rare Disease Treatments
Thursday, September 12 at 10:00 a.m. EST
Room B-318 Rayburn House Office Building
Continental breakfast will be served*
Expert presenters will include:
- Patrick J. Scannon, M.D., Ph.D., Founder, Executive Vice President and Chief Scientific Officer, XOMA Corporation
- John Crowley, Parent Advocate & CEO, Amicus Therapeutics
- Miriam O’Day, Senior Director Public Policy, Alpha-1 Foundation
- Tracy VanHoutan, Parent Advocate, Noah’s Hope, Batten Disease Support & Research Association (BDSRA)
The briefing will explore the following topics:
- Concerns about the timely implementation of the FDA Safety and Innovation Act (FDASIA) in giving rare diseases access to Accelerated Approval
- Regulatory challenges that prevent access for US patients to participate in clinical trials for rare disease treatments
- The potential elimination of the Orphan Drug Tax Credit
- H.R. 2725, the Food and Drug Administration Safety Over Sequestration (“FDA S.O.S.”) Act of 2013
For more information, please contact Julia Jenkins with Rare Disease Legislative Advocates (email@example.com or 202-803-6047), or Jenny Carey, CHI’s associate director of federal government relations and programs (firstname.lastname@example.org or 202-974-6323).
*This event is held in strict accordance with House and Senate Ethics rules. Food and refreshments of a nominal value will be provided as allowed by House Rule 25, clause 5(a)(3)(U) and Senate Rule 35.1(d)(1).