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”
Hosted by:
Ask your Members of Congress to Join the Caucus
It will only take 2 minutes: RDLA will automatically find your legislators for you. All you need to do is enter your address and add a personal note about why it’s important to you. If your legislator is already a member of the Caucus then you can send a thank you letter to your legislator instead.
Caucus Briefings:
September 14th, 2016 – Strengthening Medical Innovation in America for Rare Disease Patients
May 18th, 2016 – The NIH and FDA: Vital Agencies in the Fight Against Rare Diseases
Nov. 5th, 2015 – Precision Medicine: New Frontiers for Rare Diseases
May 21st, 2015 – 21st Century Cures Initiative: Priorities for the Rare Disease Community
Feb. 26th, 2015 – Urgent Healthcare Policy Needs of the Rare Disease Community
Sept. 17th, 2014 – Implementation of Rare Disease Provisions in FDASIA
May 7th, 2014 – Access to Care & Therapies in the New Healthcare System: A Rare Disease Perspective
Feb. 28th, 2014 – Science Behind Rare Disease Policy, Feat. Special Guest Jonny Lee Miller
