Sept. 12: Rare Disease Congressional Caucus Briefing Videos

RDCC-Horizontal-CMYKRDLA and Shire in coordination with Rare Disease Caucus Co-Chairs Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY) hosted a briefing on Thursday, September 12, 2013 

Orphans In Jeopardy:  Threats to the Development of Rare Disease Treatments 

Moderator: John Crowley, Parent Advocate & CEO, Amicus Therapeutics

Special Guest Speaker: Representative Gus Bilirakis (R-FL)
Concerns about the timely implementation of FDASIA in giving rare diseases access to Accelerated Approval

Panel Speakers:

Miriam O’Day, Senior Director Public Policy, Alpha-1 Foundation
The potential elimination of the Orphan Drug Tax Credit

Tracy VanHoutan, Parent Advocate, Noah’s Hope, Batten Disease Support & Research Association (BDSRA)
Regulatory challenges that prevent access for US patients to participate in clinical trials for rare disease treatments

Patrick J. Scannon, MD, PhD, Founder, Executive Vice President and Chief Scientific Officer, XOMA
Food and Drug Administration Safety Over Sequestration Act of 2013, H.R. 2725

Panel Discussion