EveryLife Statement on the Creation of FDA Rare Disease Innovation Hub

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.

“We are grateful for the continued leadership of Commissioner Califf, along with Dr. Peter Marks and Dr. Patrizia Cavazonni, in recognizing the unique needs of the patients we represent,” said Michael Pearlmutter, CEO of the EveryLife Foundation for Rare Diseases. “A more deliberate approach that includes a single point of contact to coordinate efforts across all Centers will be instrumental in bringing rare disease therapies to patients faster.”

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 Amy 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. Earlier this year, EveryLife Foundation submitted a report language request to the Fiscal Year 2025 Agriculture, Rural Development, Food and Drug Administration and Related Agencies Appropriations Act supported by over 100 patient advocacy organizations calling for the creation of the inter-center institute.

The EveryLife Foundation recognizes the impact of the scientific and policy leaders, combined with the thousands of rare disease community members whose experiences informed their collective advocacy. This is a positive and meaningful step forward in addressing the regulatory science challenges unique to rare disease therapy development and we look forward to working with the Agency and stakeholders to ensure that the Innovation Hub is sustained over time.

“Time is our most precious commodity in rare disease,” said Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement of EveryLife Foundation. “We are grateful to FDA for recognizing the opportunity to enhance and streamline their existing processes, centralizing and optimizing the extensive rare disease expertise across the FDA and organizing FDA rare disease resources.”

About:
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.

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