In response to the FDA’s draft guidance on Expedited Programs for Serious Conditions, Representatives Gus Bilirakis (R-FL), GK Butterfield (D-NC), Ileana Ros-Lehtinen (R-FL), and Jim Moran (D-VA) circulated a bipartisan letter in Congress calling on the FDA to fulfill the mandate to improve access to the Accelerated Approval pathway for rare diseases as required by The Food and Drug Administration Safety and Innovation Act (FDASIA). The rare disease community jumped into action, contacting their Members of Congress and urging them to sign. On September 20, 2013, the final letter was sent with 109 Congressional signatures.
The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases.
Committee on Oversight & Government Reform’s FDA Fact Checkup: Drug Development & Manufacturing Challenges, Washington, DC, December 12, 2013
FDA Public Workshop: Complex Issues in Developing Drug and Biological Products for Rare Diseases, Silver Spring, MD, January 6-7, 2014
A Rare Affair, San Francisco, CA, January 12, 2014
Lysosomal Disease Network 10th Annual WORLD Symposium, San Diego, CA, February 11-13, 2014
Rare Disease Legislative Conference and Lobby Days, Washington DC, February 255-7, 2014
Rare Disease Day at National Institutes of Health, Washington DC, February 28, 2014
Rare Disease Day, February 28, 2014