WASHINGTON, July 17,
2024 /PRNewswire/ -- 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 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. 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.
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.
For additional information,
visit everylifefoundation.org/news/. Follow us on Facebook,
Twitter, Instagram, and LinkedIn.
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SOURCE EveryLife Foundation for Rare Diseases