Bilirakis Bill to Boost Development of Cures for Rare Diseases Examined in Congressional Hearing

Washington, D.C. During today's House Health Subcommittee Legislative Hearing, Members examined the Speeding Therapy Access Today (STAT) Act of 2021 authored by Representatives Gus Bilirakis (R-FL) and G. K. Butterfield (D- NC). 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.

"I'm so glad to see the STAT Act moving forward. For me, this work is very personal because I have, many close family members, friends, and constituents who suffer with rare diseases. I've seen the hardships caused by the lack of available treatments and therapies. This is a very common problem for rare disease patients,"said Congressman Gus Bilirakis. "The targeted FDA reforms contained our bill 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."

Rare diseases are not a rare problem; more than 30 million Americans are living with one or more rare disease. Sadly, between 93% and 95% 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 Center of Excellence model has already generated positive results in other areas. Three years ago, the FDA established the first FDA Center of Excellence, focused on oncology, which has been extremely successful in bringing new cancer therapies to patients. The goal is to replicate these outcomes in the rare disease community.