Video: March 3rd Rare Disease Caucus Briefing on The Rare Disease Ecosystem

Earlier this month, the Rare Disease Congressional Caucus hosted a briefing on The Rare Disease Ecosystem: Fostering Patient Engagement & Driving Biomedical Innovation. This briefing was featured as part of the 2016 Rare Disease Week on Capitol Hill. In addition to speeches by caucus co-chairs Senators Orrin Hatch and Amy Klobuchar, Representatives Leonard Lance and Joe Crowley, Representative Gus Bilirakis made a surprise appearance.

Moderated by CEO of Raptor Pharmaceuticals Julie Anne Smith, CEO, the panelists included:

  • Janet Woodcock, M.D., Director, Center for Drug Evaluation (CDER), at the Food and Drug Administration (FDA) – “The Role of the FDA and CDER in the Drug Development Process” 
  • Emil Kakkis, M.D. PhD, President, EveryLife Foundation for Rare Diseases & CEO, Ultragenyx Inc. – “Developing Medicines for Rare & Ultra Rare Diseases”
  • Rakesh Marwah, M.D., Investment Professional, Palo Alto Investors, Clinical Faculty, Stanford University“Incentives Driving Biomedical Investment & Innovation” 
  • David Fajgenbaum, M.D., M.B.A., M.Sc., Executive Director, Castleman Disease Collaborative Network, Assistant Professor of Medicine & Associate Director, Orphan Disease Center, University of Pennsylvania – “Patient/Physician/Researcher Perspective: Driving Research & Innovation for Castleman Disease
  • Brett Felter, J.D., Assistant Attorney General, Maryland Office of the Attorney General & Becker Muscular Dystrophy Patient – “Patient Perspective: How Policy Impacts Development of Rare Disease Therapies” 

For more information on this and future caucus briefings, visit the Rare Disease Caucus website.