Maybe Obscure to You, but Life-Changing for Millions of Americans

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National Organization for Rare Disorders (NORD)

Nov 15, 2022, 11:56 ET

126 Organizations Urge Congress to Include Critical FDA Programs and Reforms in Year-End Spending Bill

WASHINGTON, Nov. 15, 2022 /PRNewswire/ -- In a letter authored by NORD and signed by 126 organizations representing or treating patients impacted by rare diseases, advocates call on congressional leaders to stand up for the more than 25 million Americans living with rare diseases by including rare disease patient priorities in an end of year legislative package.

"Millions of Americans living with rare diseases are counting on our elected leaders to finish the job to ensure the important programs and reforms that patients have fought for over the last year do not disappear," said Peter Saltonstall, NORD's President and CEO.

Last month, Congress passed HR 6833, which included a five-year reauthorization of several critical FDA user fee programs that help ensure patients gain access to essential therapies and diagnostic tools in a timely manner. However, in passing the bill, Congress broke with precedent and included only short-term reauthorizations to several items critical to the rare disease community — including the Orphan Products Grants program and the Best Pharmaceuticals for Children program.

Congress also neglected to include several key reforms that, if implemented, will help rare disease patients and their health care providers know that a therapy is truly safe and effective for them. Left behind were bipartisan supported efforts to strengthen the accelerated approval pathway, clarify and codify the scope of orphan drug exclusivity, and expand clinical trial diversity.

"We are urging Congress to do the right thing and ensure that rare disease patients from coast to coast can benefit from these programs and reforms that, taken together, can spur innovation and improve patient access to life-changing treatments and innovations," said Saltonstall.

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 330 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.

SOURCE National Organization for Rare Disorders (NORD)