Congress Introduces Legislation Intended to Speed Development of Rare Disease Therapies

Rare Daily Staff

The U.S. House and Senate introduced the Speeding Therapy Access Today Act of 2021, or STAT Act, a bipartisan bill aimed at improving the development of and access to therapies for the rare disease community.

The legislation is a result of more than a year of collaborative effort by rare disease community partners and congressional leaders. Senator Amy Klobuchar (D-Minnesota), Senator Roger Wicker (R-Mississippi), and Representatives Gus Bilirakis (R-Florida) and George Butterfield (D-North Carolina), all of whom serve as co-chairs of the Rare Disease Congressional Caucus, introduced the legislation.

“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 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.”

At the heart of the STAT Act is a provision to create a Rare Disease Center of Excellence that will address the unique challenges across rare diseases, providing solutions that can benefit therapeutic development for many communities. This approach has proven successful for cancer patients with the establishment of the Oncology Center of Excellence in 2017.

“Millions of Americans are living with one or more rare disease, the majority of which do not have FDA approved treatments,” said Senator Wicker. “Creating a Rare Disease Center of Excellence would accelerate the development of therapies for rare diseases and disorders at the FDA while putting patient input first. This would be an important step forward as we work to find cures for all diseases.”

A rare disease is defined as one that affects fewer than 200,000 people in the United States. Many rare diseases, though, effect fewer than 600 Americans. Under the STAT Act, grants and contracts would be provided to fund regulatory science and related activities to support the development of therapies to treat ultra-rare disease populations.

The legislation would inform rare disease policies and actions by creating a Rare Disease and Condition Drug Advisory Committee. The committee would advise the HHS Secretary on policies to address barriers impeding development of and access to rare disease therapies.

And, the act would also enhance coverage of drugs, biologic and gene/cell-based therapies to treat rare diseases and disorders, so payer coverage policies reflect the totality of information used by the Food and Drug Administration to determine a drug’s indicated usage and population.

“The development process for a rare disease therapy takes an average of 15 years. For the more than 30 million Americans living with rare disease, 15 years is too long. We need therapies—STAT,” said Julia Jenkins, EveryLife Foundation for Rare Diseases Executive Director.

Photo: Senator Amy Klobuchar, D-Minnesota