After more than a year of collaborative efforts by rare disease community partners and congressional leaders, the U.S. Senate and House of Representatives jointly introduced the Speeding Therapy Access Today Act of 2021 (H.R. 1730/S. 670 or STAT Act) in March 2021.* 

Introduced by Senator Amy Klobuchar (D-MN) and Senator Roger Wicker (R-MS) and Representative Gus Bilirakis (R-FL), and Representative G.K. Butterfield (D-NC), the bill is aimed at improving the development of and access to safe and effective treatments for the rare disease community.

What will the STAT Act do?

The STAT Act’s main goal is creating a Rare Disease Center of Excellence, which will expedite the development of and access to the aforementioned safe and effective therapies for the rare disease community. 

It will achieve this by:

• Maximizing interagency coordination regarding rare disease expertise
• Supporting science-based policies
• Highlighting development for ultra-rare diseases
• Enhancing coverage of drugs, biologic, and gene/cell-based therapies to treat rare diseases and disorders
• Ensuring that the intended patient populations receive timely access to approved therapies

“Thirty million Americans are impacted by one of 7,000 known rare diseases, but nearly 93 percent of these known rare diseases have no FDA-approved treatment,” said Senator Amy Klobuchar. “It is critical that we pass this legislation to increase the number of safe, effective, and affordable treatments available for people suffering from diseases. As the co-chair of the Rare Disease Congressional Caucus, I’ll keep working to prioritize the needs of rare disease patients and their loved ones.”

How will the STAT Act address barriers to treatment?

The STAT Act will inform rare disease policy by establishing a Rare Disease and Condition Drug Advisory Committee to address barriers to the development and access of treatments. 

“For me, this work is very personal because I have close family members and friends who suffer with rare diseases,” said Congressman Gus Bilirakis. “The lack of available treatments and therapies for rare disease patients is a common problem.” He added that the new legislation is intended to “provide hope to the millions of patients and families who are coping with a rare disease.”

What is considered a rare disease?

A rare disease is defined in the U.S. as one that affects fewer than 200,000 people. Some affect fewer than 600. IgA nephropathy, a form of chronic kidney disease (CKD), is one such example of a rare disease. The STAT Act would provide grants to fund research and related activities to help these patient populations.

“There are over 7,000 known rare diseases and no more than 5% have FDA-approved therapy,” stated Congressman G. K. Butterfield. “Millions of Americans are suffering avoidable delays and barriers to new therapies due to overly complex regulatory processes that can take over a decade to develop. Patients in the rare disease community are in desperate need of options and cannot wait.” 

*Every Life Foundation. (2021, Mar. 12). Congress Introduces the Speeding Therapy Access Today Act. https://everylifefoundation.org/congress-introduces-the-speeding-therapy-access-today-act/