Washington, D.C., Feb 28 (NationPress) A bipartisan panel in the U.S. Senate has criticized the Food and Drug Administration (FDA) for its delays and inconsistent standards, which, according to witnesses, are hindering access to treatments for rare diseases. They emphasized that “time is the most precious commodity” for families facing these challenges, insisting that “rare disease patients cannot wait.”

During a session of the Senate Special Committee on Aging, Chairman Rick Scott raised the critical issue of whether the FDA is fulfilling its responsibility to expedite the delivery of safe and effective treatments for individuals with rare conditions.

Scott highlighted that “time is something patients cannot afford to lose,” pointing out that “time means the ability to walk and independence.” He accused the FDA of employing “inconsistent review practices, shifting standards, and redundant, often late requests for data,” which prioritize bureaucratic procedures over patient needs.

Ranking Member Kirsten Gillibrand noted that Congress has provided ample “regulatory flexibility” to hasten the approval of rare disease therapies, including “the accelerated approval pathway” and enhanced utilization of “patient experience data and real-world evidence.” However, she expressed concern that “it’s not functioning as intended.”

Annie Kennedy from the EveryLife Foundation for Rare Diseases informed the senators that since early 2025, they have witnessed at least 23 complete response letters rejecting rare disease therapies, many of which were seeking accelerated approval. She also pointed out that advisory committee meetings for drugs and biologics have “declined by 65 percent compared to 2024.”

Kennedy expressed the community's apprehension regarding reversals in prior regulatory agreements and the diminishing chances for external experts and patients to contribute to decision-making. “We urge Congress to engage with the FDA to clarify its approach regarding accelerated approval,” she stated.

Harvard neurologist Jeremy Schmahmann appealed to lawmakers, saying, “Senators, please assist us in reforming the FDA.” He described the FDA's rejection of troriluzole for treating spinocerebellar ataxia as a denial of “a safe drug.” He noted that patients on this therapy had stabilized, emphasizing that “in this field, maintaining the same condition is success.”

Schmahmann recounted writing “six letters to FDA leadership between 2023 and 2025” without receiving a response. He warned that “300 patients stable on Troriluzole will have to discontinue the drug, leaving them distraught.” He criticized a proposed new trial as “another placebo-controlled trial that will require 5 to 8 years,” asserting, “If this occurs, patients on placebo will die.”

Bradley Campbell, CEO of Amicus Therapeutics, stressed that innovation in rare disease treatment must “adapt in speed, agility, and flexibility.” He recalled a Pompe disease patient, emphasizing that “if she could breathe on her own for just one minute,” it could significantly impact her life.

Campbell cautioned that regulatory uncertainties jeopardize the U.S. position in the global market, stating, “If we continue to foster this uncertainty... I am confident that investor dollars will relocate elsewhere.”

Cara O’Neill from the Cure Sanfilippo Foundation cited existing barriers faced by families. She explained that a gene therapy was “denied approval not due to safety or patient benefits, but over manufacturing concerns.” She argued that the FDA “could have utilized its flexibility to continue reviewing the application while addressing questions simultaneously.”

Scott reiterated that the concern was not about compromising safety standards but about whether the system is functioning with the urgency that Congress intended and that patients urgently need. “The question is not whether to ensure safety but whether the system operates with the necessary urgency,” he remarked, promising ongoing oversight from the committee.

In the U.S., rare diseases are classified as conditions affecting fewer than 200,000 individuals, yet they collectively impact over 30 million Americans. While fewer than 5 percent of rare diseases have an approved treatment, lawmakers have enacted various measures over the years, including the Orphan Drug Act and the 21st Century Cures Act, to incentivize development and facilitate accelerated approvals for serious conditions.