ICYMI: Rare Disease Community Takes Action to Strengthen and Reform Accelerated Approval 

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90 Patient Organizations Sign-on to NORD Letters to Secretary Becerra and Congressional Leaders on Strengthening the Accelerated Approval Pathway 

April 8, 2022, Washington, DC – This week, the National Organization for Rare Disorders (NORD), along with 90 other patient organizations sent letters to Health and Human Services (HHS) Secretary Xavier Becerra and Congressional leaders urging them to seize opportunities before them to protect patient access and strengthen the accelerated approval pathway.  

One letter is directed toward Congressional leadership urging them to incorporate into legislation to reauthorize the Prescription Drug User Fee Act (PDUFA) several policy recommendations to strengthen the accelerated approval.

Another letter is directed to HHS Secretary Becerra, urging him to support the efforts underway within Congress and FDA to strengthen the accelerated approval pathway and reject policies that could limit patient access to rare disease and other therapies that utilize accelerated approval. 

“Accelerated approval is vital to patients with rare diseases and other life-threatening medical conditions where no treatment currently exists,” said Heidi Ross, Acting Vice President, Policy and Regulatory Affairs, NORD. “However, NORD and the other 90 co-signing organizations believe reforms are necessary to help ensure patients have timely access to rare disease treatments that have been FDA approved through the accelerated approval pathway. NORD looks forward to working with Congress and HHS to ensure changes to the accelerated approval pathway ultimately benefit the rare disease community.” 

To learn more about NORD’s policy work, visit: https://bit.ly/Policy-Issues. 

About the National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit rarediseases.org.