With so much bad news in the world, it is worth pausing for one encouraging development: Marty Makary finally resigned as commissioner of the Food and Drug Administration last week.
Makary’s tenure at the FDA was marred by internal scandals, forced resignations, dreadful morale, and record staff turnover. More important, he actively sandbagged President Trump’s push to expand clinical trials for rare diseases through the aptly named “right-to-try” framework.
Trump’s next appointee should restore the spirit of right to try and make safe, effective treatments available to children as quickly as possible.
The idea behind right to try is straightforward. Patients with rare conditions, especially those for whom conventional medicine has failed, should have the freedom to pursue experimental treatments that have not yet received full FDA approval. Families fighting the clock have little left to lose. Government should not stand between them and a potentially lifesaving breakthrough.
Makary did.
Members of the MPS community sent more than 10 letters asking Makary for a meeting. They got a form letter in return. Sen. Ron Johnson (R-Wis.) later announced an investigation into the FDA’s denials. Makary’s agency responded by claiming approvals were already “at their peak.” The Wall Street Journal took notice of the FDA’s foot-dragging last year, yet the agency kept rejecting relevant rare-disease treatments in early 2026, including RGX-121 and drugs from Biohaven and Saol Therapeutics.
That stonewalling forced families to escalate.
In March, more than 100 mothers and other advocates staged a mock funeral outside FDA offices. Dressed in black and carrying a real coffin, they sought to draw attention to a group of rare metabolic disorders known as mucopolysaccharidoses. These disorders can show up as mild symptoms such as depression or hyperactivity, or as devastating conditions such as heart disease and skeletal abnormalities.
Many MPS disorders still have no approved treatments, even though they can severely diminish children’s quality of life or kill them outright. The FDA’s regulatory process serves a legitimate purpose. But when a bureaucracy grows so rigid, self-protective, and arrogant that it blocks desperately ill children from access to promising therapies, it stops functioning as a safeguard and starts functioning as a death sentence.
Mark Dant of the Ryan Foundation told Newsweek that some of these drugs were denied because of the FDA’s institutional “dislike” of the accelerated-approval pathway. “For decades we waited for science to find our tomorrows,” he said. “Now it has, and bureaucrats within the agency we pay for are keeping those treatments from our children. We know they are there. … We just cannot reach them.”
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Valerie Plesch/Bloomberg/Getty Images
Makary’s resignation will not undo the damage. But it does create an opening. We may not yet know what the FDA’s next leadership will look like, but Trump’s appointee should restore the spirit of right to try and make safe, effective treatments available to children as quickly as possible.
Across the world, in the nation of Georgia, parents have staged a protest lasting more than 500 consecutive days, maintaining a round-the-clock presence outside the main government building in Tbilisi. They are willing to risk everything to give their children the best chance at life. Americans should not have to camp outside federal offices for 500 days to get their government to listen.
The new FDA leadership must explain denials of right-to-try clinical trials with enough specificity that sponsors and families understand what evidence could change the decision. Patient and caregiver testimony should shape decisions early, not get folded in at the end as a token gesture. And Congress must demand transparency without turning each drug review into a partisan circus.
Children’s lives are not bargaining chips. The FDA exists to serve the public, not to protect its own bureaucracy from embarrassment. If Makary’s departure opens the door to that truth, families battling ultra-rare diseases may finally have reason to hope.
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