WASHINGTON, June 8, 2022—Today, the U.S. House of Representatives passed by a vote of 392-28 the Food and Drug Amendments of 2022 (H.R. 7667), legislation with significant implications for millions of Americans living with rare diseases. The bipartisan bill is an important step forward in reauthorizing the FDA’s medical product user fee programs, helps strengthen the accelerated approval pathway, and protects the intent of the Orphan Drug Act.
“On behalf of millions of Americans living with rare diseases, we commend House lawmakers for working in a bipartisan way to pass the critically important Food and Drug Amendments of 2022,” said Heidi Ross, Vice President, Policy & Regulatory Affairs, National Organization for Rare Disorders (NORD®). “It is clear from this vote that our elected officials have heard our community’s calls for action, and they recognize the importance of these user fee acts in helping to address the urgent needs of their constituents who are affected by rare diseases.”
Ross added: “We wish to convey gratitude to the House for the inclusion of provisions in Section 804 to strengthen the accelerated approval pathway to help people with rare diseases and other life-threatening medical conditions where no treatment currently exists; and Section 812, which clarifies the intent of the Orphan Drug Act, ensuring appropriate incentives are maintained to encourage robust rare disease drug development.”
The FDA largely relies on user fees authorized by Congress to ensure the timely review of drug and medical device applications. Without the reauthorization of these user fee programs, a significant number of the FDA’s drug, biologic and device reviewers would be laid off, and the vital review of innovative therapies would be substantially impaired.
The passage of H.R. 7667 marks nearly two years of advocacy and policy work by NORD in support of the Prescription Drug User Fee Act (PDUFA) reauthorization that has included submitting testimony and comments to Congress, a white paper on the accelerated approval pathway and recommendations to strengthen it, call-to-action campaigns, a letter to House leadership signed by 91 advocacy organizations and a recent statement for the record.
“These reforms are necessary to help ensure rare disease patients have timely access to treatments that have been FDA approved,” said Ross. “We thank the leadership of the House Energy and Commerce Committee and members of the House of Representatives for moving forward with these critical funding mechanisms and provisions to support patient access to innovative therapies.”
H.R. 7667 now moves to the U.S. Senate, where similar legislation, S. 4348, the Food and Drug Administration Safety and Landmark Advancements Act of 2022, is scheduled to be considered in the Senate Committee on Health, Education, Labor and Pensions next week.
Follow online at #RareAction for updates and keep up with NORD’s recent policy statements here.
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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.