U.S. Rep. Gus Bilirakis, R-Fla., has introduced a proposal to have the U.S. Food and Drug Administration (FDA) speed up its processes to approve therapies for rare diseases.
Last week, Bilirakis brought out the “Speeding Therapy Access Today (STAT) Act” with U.S. Rep. G. K. Butterfield, D-NC, as a co-sponsor.
“This bipartisan, bicameral legislation was developed with the input of the rare disease community, and is aimed at improving the development of and access to therapies for the rare disease community,” the Florida congressman’s office noted. “Rare diseases are not a rare problem; more than 30 million Americans are living with one or more rare disease. idly, between 93 percent and 95 percent of the more than 7,000 known rare diseases have no U.S. Food and Drug Administration (FDA)-approved therapy. Traditional regulatory processes have become more complex involving combinations of therapies, genomics, novel diagnostic tests, multi-systemic diseases, small patient populations, and precision medicine. As a result, numerous parts of the regulatory system need to cohesively work together. The development process for a rare disease drug currently takes an average of 15 years, which leaves rare disease patients and their families waiting without access to life-changing or even life-saving treatments. Further complicating the situation is that when new therapies for rare diseases are finally approved under the current structure, patients often face unnecessary delays and barriers to access, resulting in avoidable health deterioration.
“The STAT Act will tackle these issues by enacting targeted, impactful, and attainable policy reforms at the FDA to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate patient access to such therapies. Specifically, the STAT Act 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 actions by creating a Rare Disease and Condition Drug Advisory Committee, fund regulatory science and related activities to support development of therapies to treat very small rare disease populations, and strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans through enhanced FDA and Centers for Medicare and Medicaid Services coordination, proactive engagement of payers, and specific actions intended to strengthen Medicare and Medicaid beneficiary access to novel therapies,” the congressman’s office added.
Bilirakis showcased the bill on Monday.
“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,” said Bilirakis. “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 percent 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,” said Butterfield. “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.”
The bill was sent to the U.S. House Energy and Commerce Committee.
Over in the U.S. Senate, U.S. Sen. Amy Klobuchar, D-Minn., introduced the companion bill last week with U.S. Sen. Roger Wicker, R-Miss., as the co-sponsor.
“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,” 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.”
Klobuchar’s bill was sent to the U.S. Senate Health, Education, Labor and Pensions Committee.
The bill has the support of the EveryLife Foundation for Rare Diseases.
Reach Kevin Derby at firstname.lastname@example.org.