The EveryLife Foundation for Rare Diseases applauded a bipartisan letter led by the Congressional Rare Disease Caucus to Food and Drug Administration (FDA) Commissioner Robert Califf that urges the FDA to identify and address gaps in rare disease activities.
Led by Congressional Rare Disease Caucus Chairs Senators Roger Wicker (R-MS) and Amy Klobuchar (D-MN) and Reps. Gus Bilirakis (R-FL) and Doris Matsui (D-CA), the letter to Commissioner Robert Califf was signed by 25 members of Congress.
In the letter, members recognized the progress that has occurred in rare disease therapy development during the 40 years since the Orphan Drug Act was enacted into law, including the approval of more than 1,000 orphan drug designations during this time. But the members also recognized that most rare diseases lack any FDA-approved therapies and noted that “there remains significant uneven application of rare disease policies, guidance, and expertise” at the agency.
The letter asks Commissioner Califf to form a task force to answer several specific questions and to report to Congress by the end of 2024 to help inform future policymaking.
“The rare disease community recognizes the transformational impact the ODA has had on rare diseases and recognizes the commitment of many FDA centers, offices, and review divisions to supporting this work while applying the Agency’s same rigorous standards for safety and efficacy. But at the same time, we believe more can be done to standardize this commitment to rare diseases across all FDA centers, offices and review divisions to ensure an even application of these important policies and protocols,” said Frank Sasinowski, Chair of the EveryLife Foundation Board of Directors and Rare Disease Regulatory Policy Expert.
EveryLife Foundation is encouraged by FDA’s recent efforts to establish additional infrastructure focused on rare disease regulatory challenges and applauds the Agency for applying the flexibility granted to FDA in the 1997 Food and Drug Administration Modernization Act in several recent approvals that represent the first available treatments for challenging rare diseases like ALS, and Friedreich’s ataxia.
The rare disease community is grateful for the Congressional leaders who met with more than 600 rare disease advocates from across the nation during Rare Disease Week and reflected the unmet need and therapeutic development opportunity within rare disease into the letter.
“We applaud the chairs of the rare disease caucus for understanding the urgency of our rare disease community. By sending this letter and urging FDA to undertake a thorough review of its rare disease policies and procedures, Congress has pointed to key recommendations for regulatory enhancements so that all rare disease communities can benefit from agency expertise and consistency of decision making, regardless of the Center or division overseeing their programs,” said Annie Kennedy, Chief of Policy, Advocacy and Patient Engagement for the EveryLife Foundation. “Time is the most precious commodity for our rare disease community. We need a regulatory system that helps facilitate the translation of today’s remarkable scientific and technology advances into better outcomes for this generation of rare disease patients.”
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What is an FDA task force and why do we need one for rare disease? Click here to learn more
