The EveryLife Foundation for Rare Diseases extends its gratitude to Representatives DeGette and Bucshon for their dedication to enhancing the 21st Century Cures Act. Their commitment to a patient-centered healthcare system is vital for advancing research, improving treatment options, and supporting those affected by rare diseases.
The 21st Century Cures Act has been a significant milestone, reshaping the therapeutic landscape for rare diseases. As the 10-year anniversary approaches, it is essential to commemorate the progress made and continue pushing for advancements. Despite over 1,200 approved indications for rare diseases, 95% of the over 10,000 known rare diseases still lack treatments. The recent establishment of the FDA Rare Disease Innovation Hub marks a crucial step, but further critical support is needed to address the unique challenges in developing and accessing treatments for rare diseases.
The EveryLife Foundation has submitted a brief response to the RFI, highlighting relevant provisions of Cures 2.0 that should carry forward and ongoing policy needs that should be incorporated into a Cures 3.0 package.
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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.
