The FDA has been in hot water for over a year — blocking rare disease, cancer, and other treatments from patients who are running out of time. That leadership is now gone. Vinay Prasad resigned as CBER director, and Commissioner Marty Makary was fired.
But what remains is an agency in chaos that still isn’t serving the public.
I have spent my career fighting for limited government and individual liberty, from lobbying for human rights to working at the National Right to Work Committee. I celebrated along with millions of other Americans when President Donald Trump signed the landmark Right to Try legislation in his first term, giving Americans with terminal illness the right to fight for their lives and make their own treatment decisions.
It was proof that Washington can still do the right thing. His administration has renewed that commitment to patients, but the FDA’s advisory committees are undermining it.
These panels of outside experts are supposed to give the agency independent, informed guidance on new treatments — especially experimental and emerging ones. Instead, they have become a bureaucratic layer that slows approvals, lacks transparency, and simply does not benefit the public. For patients fighting rare diseases that are too often fatal, these committees don’t just fall short. They stand in the way.
A doctor with broad clinical knowledge can offer something useful on a general medical question. But the idea that the same generalist can meaningfully evaluate a treatment for Duchenne Muscular Dystrophy — which impacts almost exclusively boys, and only a few dozen new patients per year — is not rigorous. It is a failure of design. Too often, committee members appear more focused on advancing their own careers than on following best practices, weighing patient input, or applying scientific evidence. Inaction becomes easier than approval.
That’s not public service. That’s bureaucracy protecting itself.
What patients need are committees that accept real-world evidence, understand the ethics of small-population research, and apply a risk tolerance calibrated to the actual stakes. For a child with Duchenne or Huntington’s, the risk of not approving a therapy is often greater than the risk of approving one. Advisory committees that treat inaction as the safe option are not protecting patients. They are abandoning them — and betraying the promise of Right to Try.
This is not a partisan issue. Families of children with Duchenne, Huntington’s, and Sanfilippo syndrome showed up to a Senate hearing in February 2026 in wheelchairs, with tears, and with nothing left to lose. The response was bipartisan. Senator Ron Johnson (R-Wis.) called out the FDA’s cruelty directly. Senator Rick Scott (R-Fla.), chairing the Special Committee on Aging, put the hearing together with Ranking Member Kristen Gillibrand (D-N.Y.). Scott condemned the FDA’s “inconsistent review practices, shifting standards, and redundant data requests” blocking access to safe and effective therapies for the more than 30 million Americans living with a rare disease.
Within weeks of those hearings, Makary and Prasad were gone.
Two resignations don’t fix a culture. Real advisory committee reform means appointing disease-specific specialists instead of generalists, mandating patient and family representation, and accepting real-world evidence rather than narrow clinical trial models that are ethically indefensible for small populations. It means committees that understand they are being called upon to make life-and-death decisions — not career moves. The General Accounting Office found that FDA advisory committees have failed to disclose financial conflicts of interest for over thirteen years, despite a congressional mandate to do so. That is the definition of a broken institution.
Conservatives believe in a government that is small, accountable, and effective. FDA advisory committees are none of those things. President Trump promised to return the power of medical decision-making to individual patients — the same principle behind Right to Try. That promise has bipartisan support, moral clarity, and the backing of science. Advisory committees that stand in the way through bureaucracy, ideology, or indifference should not survive this administration. The next FDA commissioner must make sure they don’t.
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