March 3 Panel on Rare Disease Ecosystem
Tomorrow at noon, the Rare Disease Congressional Caucus will convene a panel of patient advocates, regulatory leaders, and biomedical innovators at the Capitol Visitor Center as part of Rare Disease Week on Capitol Hill.
The bipartisan Caucus was recently expanded to encompass the House and Senate. Co-chairs Reps. Leonard Lance (R-NJ) and Joe Crowley (D-NY) as well as Sens. Orrin Hatch (R-UT) and Amy Klobuchar (D-MN) are scheduled to provide remarks.
The panel will be moderated by Julie Anne Smith, CEO of Raptor Pharmaceuticals. Dr. Janet Woodcock, Director of the Center for Drug Evaluation (CDER) at the Food and Drug Administration (FDA) will discuss the regulatory perspective. Dr. Emil Kakkis, President of the EveryLife Foundation for Rare Diseases and CEO of Ultragenyx, will provide perspective on drug development and public policy. Dr. Rakesh Marwah of Palo Alto Investors and Stanford University will offer his perspective as a physician and investor. University of Pennsylvania rare disease researcher and Castleman Disease Collaborative Network Co-Founder and Executive Director Dr. David Fajgenbaum will speak as a physician, researcher and patient. The panel will also feature Brett Felter, J.D., a Becker Muscular Dystrophy patient and Assistant Attorney General for the state of Maryland.
More than 200 rare disease advocates from across the country are in Washington, D.C. to learn about policy issues facing the community and meeting with their elected representatives as part of Rare Disease Week on Capitol Hill. The week is coordinated by Rare Disease Legislative Advocates, a program of the EveryLife Foundation for Rare Diseases, which is dedicated to accelerating the pace of biomedical innovation for rare disease patients.
Rare diseases affect approximately 30 million Americans. Of the 7,000 known rare diseases, 95% have no FDA-approved treatment and rare disease patients in the United States wait an average of seven years for an accurate diagnosis.
Source: Business Wire