FDA Faces Criticism Over Rare Disease Treatment Delays

Advocates say the agency's culture is failing patients with life-threatening conditions

Mar. 31, 2026 at 12:05am

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The Food and Drug Administration has faced growing criticism over its handling of rare disease treatments, with patients and advocates accusing the agency of unnecessary delays in approving potentially life-changing drugs. Recent leadership changes, including the departure of a top official, have done little to address the underlying cultural issues within the FDA, they say.

Why it matters

Patients with rare, terminal diseases and their families are desperate for access to any treatments that could extend or improve their quality of life. Delays in approving these therapies can have devastating consequences, as the conditions often progress rapidly. Advocates argue the FDA's approach reflects a dismissive attitude toward the rare disease community.

The details

The article cites several examples of the FDA's perceived mistreatment of rare disease patients. This includes the recent departure of Dr. Vinay Prasad, a top FDA official who was accused of blocking access to new drugs, as well as a controversial social media post by an HHS spokesperson deriding Huntington's disease advocates as 'the swamp.' Patients and their families have voiced growing concerns about the agency delaying promising therapies, arguing their advocacy is driven by necessity, not politics or profit.

  • In the past year, patients and advocates have increasingly voiced concerns about FDA delays in approving rare disease treatments.
  • Dr. Vinay Prasad recently departed the FDA, where he had been accused of blocking access to new drugs.
  • An HHS spokesperson's derogatory social media post about Huntington's disease advocates was deleted but sparked outrage.

The players

Dr. Vinay Prasad

A former top official at the FDA's Center for Biologics Evaluation and Research, who was accused of blocking access to new drugs for rare disease patients.

Andrew Nixon

The Deputy Assistant Secretary for Media Relations at the U.S. Department of Health and Human Services, who referred to rare disease advocates as 'the swamp' in a now-deleted social media post.

Senators Rick Scott and Ron Johnson

Members of the U.S. Senate Special Committee on Aging who have shone a spotlight on the struggles facing rare disease patients and their families.

President Donald Trump

The former president who supported the Right to Try initiative, recognizing the importance of giving terminally ill patients access to promising treatments.

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What they’re saying

“Patients and their advocates are not the swamp — and no one can drain the love that drives us to fight for hope and a better life for people living with rare diseases.”

— Melissa Ortiz, Advocate and former HHS employee

“When time is running out, access to promising treatments can mean everything. The patients with those diseases and their families, not a bureaucrat in Washington, should be able to decide whether a drug is too 'dangerous' to take.”

— President Donald Trump

What’s next

Senators Scott and Johnson have vowed to continue pressing the FDA for greater accountability and responsiveness to the needs of rare disease patients. Advocates hope the departure of Dr. Prasad and growing public scrutiny will lead to a cultural shift within the agency.

The takeaway

The FDA's perceived dismissive attitude toward rare disease patients and their families has eroded trust and highlighted the urgent need for the agency to prioritize the well-being of these vulnerable communities. Meaningful change will require not just leadership transitions, but a fundamental shift in the agency's approach and priorities.