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# Advocates Urge Congress to Rein In FDA Review Uncertainty
- URL: https://www.fdaweb.com/advocates-urge-congress-to-rein-in-fda-review-uncertainty/
- Published: 2026-02-26T12:00:00.000Z
- Updated: 2026-09-14T13:34:26.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5160733

A leading rare disease advocacy group on Thursday urged lawmakers to increase oversight of the Food and Drug Administration, warning that inconsistent use of regulatory flexibility and a drop in advisory committee meetings are creating uncertainty that could delay life-saving therapies.

Testifying before the [U.S. Senate Special Committee on Aging](https://www.aging.senate.gov/download/02/26/2026/testimony%5Fschmahmann-022626pdf?ref=fdaweb.com), **Annie Kennedy**, chief mission officer of the EveryLife Foundation for Rare Diseases, said recent agency actions have “shifted momentum” for the more than 30 million Americans living with rare diseases.

Committee chairman **Rick Scott** (R-FL) questioned whether FDA is fulfilling congressional intent to expedite safe and effective therapies for serious and life-threatening rare conditions. Scott pointed to bipartisan reforms such as the 21st Century Cures Act, which encourages FDA to use tools like real-world evidence and regulatory flexibility to accelerate rare disease drug development. Nearly a decade later, however, he said patients, physicians and drug developers report that the system is “not working as Congress intended.”

Scott acknowledged efforts by FDA commissioner **Marty Makary** to address what he described as longstanding agency challenges, but said advocates continue to cite inconsistent review practices, shifting standards and late-stage data requests that create uncertainty for sponsors. He said he hopes the hearing will help lawmakers identify ways to improve accountability, efficiency and transparency in the FDA’s review process for rare disease therapies.

Kennedy warned that recent regulatory trends are undermining predictability. Since the start of 2025, at least 23 complete response letters declining approval for rare disease therapies have been issued, many involving applications under the accelerated approval pathway. Some letters, she said, suggest hesitation to rely on surrogate endpoints, natural history data, external controls, and real-world evidence—tools long considered essential in small patient populations where traditional randomized, placebo-controlled trials may be infeasible or unethical.

She also pointed to a 65% decline in advisory committee meetings for drugs and biologics in 2025 compared with the prior year, reducing opportunities for external scientific input. In some cases, meetings expected to review rare disease products were canceled before negative regulatory decisions were issued, she said.

Kennedy called on Congress to conduct oversight of how the FDA is applying the accelerated approval pathway to rare disease therapies, address what she described as inconsistent use of regulatory flexibility, and encourage the agency to resume convening advisory committees for complex reviews.

She also urged lawmakers to strengthen and better resource the FDA’s Rare Disease Innovation Hub and to establish a standing Rare Disease and Condition Advisory Committee to provide broader stakeholder input on regulatory policy.

While acknowledging that the agency has implemented rare disease provisions in recent user fee legislation, Kennedy said their benefits have yet to be fully realized.

“At a time when advances in science have put life-altering treatments within reach,” she told lawmakers, uneven application of regulatory tools risks slowing investment, stalling development pipelines, and leaving patients without options.

For families facing progressive, debilitating conditions, she said, time remains the most precious — and scarce — resource.