The EveryLife Foundation for Rare Diseases commends the Food and Drug Administration (FDA) on today’s announcement of its intention to create a Rare Disease Innovation Hub. Co-chaired by CDER Director, Dr. Peter Marks, and CBER Director, Dr. Patrizia Cavazzoni, the Hub will directly engage patients and the public to inform its strategic agenda. Most importantly, the Hub will provide a single point of contact within the Agency, a newly created position in the Director of Strategic Coalitions, to ensure streamlined engagement, consistency, and coordination across both Centers.
Since convening a Scientific Workshop on the topic in 2018, EveryLife Foundation has been advocating for the creation of an inter-center institute for rare diseases at the FDA, i.e. a Center for Excellence for Rare Diseases, to serve as the FDA’s coordinating office for engagement with rare disease stakeholders across all therapies including drugs, cell and gene therapies, and medical devices. These efforts were bolstered by the leadership of the Congressional Rare Disease Caucus and its co-chairs, Representatives Gus Bilirakis (R-FL) and Doris Matsui (D-CA) and Senators Ruth Klobuchar (D-MN) and Roger Wicker (R-MS), through their introduction of the Speeding Therapy Access Today Act in the 117th Congress and a powerful letter to the FDA requesting the formation of a rare disease task force in 2022.
