WASHINGTON - U.S. Senator Amy Klobuchar (D-MN), Senator Roger Wicker (R-MS), Representative Gus Bilirakis (R-FL), and Representative G. K. Butterfield (D-NC), leaders of the Rare Disease Congressional Caucus, introduced bipartisan, bicameral legislation to enact policy reforms at the Food and Drug Administration (FDA) to accelerate the development of therapies for rare diseases and disorders.

The Speeding Therapy Access Today (STAT) Act will improve access to therapies for the rare disease community, including patients living with an ultra-rare disease, by promoting coordination within the government to advance science-based policies and ensure the intended patient populations ultimately receive access to approved therapies. 

“30 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,” Klobuchar said. “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.”

“Millions of Americans are living with one or more rare disease, the majority of which do not have FDA approved treatments,” Wicker said. “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.”

“For me, this work is very personal because I have close family members and friends who suffer with rare diseases. The lack of available treatments and therapies for rare disease patients is a common problem,” Bilirakis said. “The targeted FDA reforms contained in this legislation will have a meaningful impact on the acceleration of treatment development and will provide hope to the millions of patients and families who are coping with a rare disease.”

“There are over 7,000 known rare diseases and no more than 5% have FDA-approved therapy. 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,” Butterfield said. “I’m proud to join my co-chairs of the Congressional Rare Disease Caucus in introducing the Speeding Therapy Access Today (or STAT) Act to accelerate development and access to potentially life-saving rare disease therapies.”

“On behalf of the more than 30 million Americans living with a rare disease, the EveryLife Foundation for Rare Diseases enthusiastically endorses the STAT Act and stands ready to help advance this impactful legislation,” said Julia Jenkins, EveryLife Foundation for Rare Diseases Executive Director. “As co-chairs of the Rare Disease Caucus, and now as co-leads of the STAT Act, Senators Klobuchar and Wicker and Representatives Bilirakis and Butterfield have been unwavering champions for the rare disease community. We are grateful for their leadership, and we look forward to working with them to ensure their innovative vision and approach to catalyzing treatments and cures for Americans with rare diseases becomes a reality.”   

Specifically, the bill will:

  • Improve rare disease coordination, stakeholder engagement, and policy development within FDA by expanding existing authority to create a Rare Disease Center of Excellence. 
  • Inform rare disease policies and the development of innovative approaches to drug approval through a Rare Disease and Condition Drug Advisory Committee.
  • Fund the development of best practices and research to support the approval of therapies to treat very small populations.
  • Ensure a broader population of rare disease patients gain access to FDA-approved drugs by ensuring public and private payer coverage align with science through a Rare Disease Therapy Access Program. 

Klobuchar, a leader in efforts to boost innovation and provide safe options in the treatment of rare diseases, has consistently called for increased funding for the National Institutes of Health and medical research to fuel the next generation of biomedical breakthroughs such as the treatment of rare diseases. Last month, she and Wicker reintroduced the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act to provide patients and advocates the ability to play a larger role in the FDA’s benefit-risk framework for drug approval.

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