Washington, February 28 – A bipartisan US Senate panel criticized the Food and Drug Administration (FDA) for delays and shifting standards that witnesses said are hindering treatments for rare diseases, warning that “time is the most precious commodity” for families and that “patients with rare diseases cannot wait.”
At a hearing of the Senate Special Committee on Aging, Chairman Rick Scott said the central question was whether the FDA is doing “everything Congress intended it to do to quickly get safe, effective treatments to patients with rare diseases.”
Scott said patients “cannot afford to lose time,” adding that “time means the ability to walk; time means independence.” He accused the agency of “inconsistent review practices, shifting standards, and redundant, often late-appearing data requests” that put “bureaucratic processes ahead of patients.”
Ranking Member Kirsten Gillibrand said Congress had provided “significant regulatory flexibility” to speed rare disease therapies, including “the accelerated approval pathway” and greater use of “patient experience data and real-world evidence.” But she added, “it’s not working as it should.”
Annie Kennedy of the EveryLife Foundation for Rare Diseases told senators that since early 2025, “we have seen at least 23 complete response letters declining to approve rare disease therapies,” many under accelerated approval. She also noted that advisory committee meetings for drugs and biologics “declined by 65 per cent compared to 2024.”
Kennedy said the community was concerned about reversals in prior regulatory agreements and fewer opportunities for outside experts and patients to inform decisions. “We ask that Congress engage with the FDA to clarify its approach to accelerated approval,” she said.
Harvard neurologist Jeremy Schmahmann urged lawmakers: “Senators, please help us fix the FDA.” He described the rejection of troriluzole for spinocerebellar ataxia as the denial of “a safe drug.” He said patients on the therapy had stabilized, adding, “in this business, staying the same is success.”
Schmahmann said he wrote “six letters to FDA leadership between 2023 and 2025” and “never received a response.” He warned that “now 300 patients stable on Troriluzole will have to discontinue the drug, and they are distraught.” Calling a proposed new trial “another placebo-controlled trial that will take 5 to 8 years,” he said, “If this happens, patients on the placebo will die.”
Bradley Campbell, chief executive of Amicus Therapeutics, said that rare disease innovation must “adapt in terms of speed, agility, and flexibility.” Recalling a Pompe disease patient, he said what would be most meaningful was “if she could breathe on her own for just one minute,” adding, “Just that 60 seconds of her own breath could make the difference between her life and her death.”
Campbell warned that regulatory uncertainty threatens US competitiveness. “If we continue to create this uncertainty… I am confident that those investor dollars would go elsewhere,” he said.
Cara O’Neill of the Cure Sanfilippo Foundation said families were seeing barriers “right now.” She said a gene therapy was “denied approval not because of safety or how the children were benefiting, but because of questions about manufacturing.” She argued that the FDA “could have used its flexibility to continue reviewing the application while addressing questions in parallel.”
Scott said the issue was not about lowering safety standards but about whether the system was moving with urgency. “The question is not whether to protect safety; it is whether the system is moving with the urgency that Congress intended and that patients require,” he said, adding that the committee would continue its oversight.
Rare diseases in the US are defined as conditions affecting fewer than 200,000 people, but collectively impact more than 30 million Americans. While fewer than 5 per cent of rare diseases have an approved treatment, lawmakers over the past decades have enacted measures such as the Orphan Drug Act and the 21st Century Cures Act to incentivize development and allow accelerated approvals for serious conditions.