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Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs:

Representative Leonard Lance (R-NJ), Representative Joe Crowley (D-NY), Senator Orrin Hatch (R-UT), and Senator Amy Klobuchar (D-MN) will host a briefing on:

The Rare Disease Ecosystem: Fostering Patient Engagement & Driving Biomedical Innovation

Thursday, March 3rd 2016

Capitol Visitor Center (CVC) Auditorium
East Capitol St NE & First St SE
Washington, DC 20004 

Congressional Co-Chair Statements – 12:00pm – 12:30pm:

Representative Leonard Lance (R-NJ)
Representative Joe Crowley (D-NY)
Senator Orrin Hatch (R-UT)
Senator Amy Klobuchar (D-MN)

Lunch Briefing: 12:30pm – 1:30pm

Moderator: Julie Anne Smith, CEO, Raptor Pharmaceuticals

  • 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” 

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