We are encouraged to hear that, following Dr. Peter Marks’ recent remarks at the Drug Information Association (DIA) conference, the FDA is contemplating the establishment of new infrastructure for rare diseases which was referred to by Dr. Marks as a ‘rare disease hub’. This development appears to align with elements of the cohesive and structured approach our rare disease community has been advocating for through the EveryLife Foundation. Since our initial discussions at the 2018 Scientific Workshop, our subsequent white paper in 2022, and most recently, in report language requesting the creation of an inter-center institute, we’ve been dedicated to the vision of an FDA Center of Excellence for Rare Diseases. Our efforts, bolstered by the leadership of the Congressional Rare Disease Caucus 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 taskforce in 2022, have aimed to catalyze such an initiative.
We are grateful for the leadership of Commissioner Califf, along with Drs. Marks and Cavazzoni, for their recognition of the need for a more coordinated approach to rare disease therapy development. As a community, we are eager to work with the agency to ensure any new infrastructure does not merely serve as a symbolic gesture, but rather is a robust inter-center framework that facilitates consistency, transparency, and coordination of rare disease experts. The effectiveness of this hub in truly serving the rare disease community will hinge on the thoroughness of its foundational documents and its operational guidelines. Our collective goal is to see this infrastructure become a genuine opportunity to enhance the FDA’s mission to support those affected by rare diseases.
About EveryLife Foundation for Rare Diseases:
EveryLife Foundation for Rare Diseases is a nonprofit, nonpartisan organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. The Foundation works to improve the lives of the millions of Americans suffering from rare diseases by advocating for policies that foster innovation and remove barriers to life-saving treatments. Founded in 2009 and headquartered in Washington, DC, the EveryLife Foundation for Rare Diseases has programs throughout the United States. To learn more, visit EveryLifeFoundation.org.
