To Top

Nov. 15, 2022

TOPIC: Press Releases, Featured News, Advocacy

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

Posted by Rohan Narayanan

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

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)
With a 40-year history of advancing care, treatments and policy, the National Organization for Rare Disorders (NORD) is the leading and longest-standing patient advocacy group for the 25-30 million Americans living with a rare disease. NORD, a 501(c)(3) nonprofit, is dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more than 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services. For more information, please visit