Bilirakis Introduces Bipartisan Bill Improving Drug Development Against Rare Diseases

The fight against rare diseases has been slow due to several factors relating to regulations, something Representatives Gus Bilirakis (R-FL) and Doris Matsui (D-CA) seek to amend in recent bipartisan legislation easing the regulatory process between the Food and Drug Administration (FDA) and rare disease specialists so that treatments can be developed more efficiently.

As Rep. Bilirakis explained in his press release, 90% of rare diseases do not have FDA-approved treatment because FDA staff cannot adequately focus on specific illnesses. At the same time, non-FDA rare disease specialists often cannot serve on the FDA's advisory committees due to conflicts of interest.

Thus, the Scientific External Process for Educated Review of Therapeutics (EXPERT) Act introduced by Rep. Bilirakis and Rep. Matsui seeks to break down these bureaucratic barriers by formalizing the Externally-Led Scientific-Focused Drug Development (EL-SFDD) meeting system within the FDA.

EL-SDFDD meetings would be held quarterly between medical experts, drug sponsors, scientific organizations, and patient advocates to discuss opportunities to improve treatment development, devise new clinical trial designs, and agree on endpoints better to meet the unmet needs of rare disease patients.

"We've repeatedly seen that tangible progress can be achieved when all stakeholders are brought together and united in a common purpose to share information, brainstorm, and develop solutions. It is important that we use this same approach when tackling the challenges facing the rare disease community, and our bipartisan legislation is the first step in making that happen.  I am eager to see the positive results that enhanced collaboration will yield, and I urge my colleagues to join me in expediting passage of this important bill," said Bilirakis.

"I am grateful for the steps the FDA has taken to advance their engagement with rare diseases, but there is more work to be done to ensure every rare disease patient has the hope of a successful therapy. We need more formalized processes for engagement to balance the urgent needs of rare disease patients, who overwhelmingly lack treatment options, with the mission of the FDA to ensure every medication in American families' drug cabinets is safe and effective. My Scientific EXPERT Act will bring rare disease experts and FDA reviewers together to share their knowledge and have productive conversations about how to move rare disease therapies forward," Matsui added.