The Food and Drug Administration is being called on to adopt a more patient-centered approach when approving treatments for rare diseases, according to an opinion piece published on July 13. The article highlights concerns from patients, families, and physicians that the agency may be losing sight of its mission to serve people living with rare conditions.
A key issue raised is the absence of advisory committee meetings for nine months starting in mid-2025 under former FDA leadership. The author said this pause created uncertainty and delayed important discussions about emerging therapies. While newly commissioned advisory committee meetings are seen as a positive step, the article argues that more must be done to ensure these panels include patient perspectives alongside medical and scientific experts.
The writer, who has experience in Duchenne muscular dystrophy research and advocacy, said that including patients and their families in advisory committees provides essential insights: “That perspective is provided best by patients, their families and other patient advocates, but too often FDA adcomms are missing those voices.” The article also notes that decisions before these committees often involve weighing incomplete data and uncertain outcomes against the urgent needs of patients with life-limiting diagnoses.
Standard approval pathways were designed for common diseases with large populations, but rare diseases differ significantly. Clinical trials may only involve dozens or hundreds of participants due to recruitment challenges. The author emphasizes that expertise in general medicine does not always translate into understanding specific rare disorders or appreciating risk tolerance unique to these communities.
The piece calls for reforms ensuring that rare disease advocates have a seat at the table during regulatory reviews. It states: “Reforms should not weaken scientific standards but strengthen the quality of decision-making by ensuring that the right experts are in the room—including patients themselves—and that the realities of rare disease are fully understood.”
Quoting physician Peter Diamandis, it concludes, “The most unethical thing in medicine is the patient who died waiting for a treatment that existed but wasn’t approved yet.”
