For the past year, the Food and Drug Administration has done the exact opposite of what it promised. Leaders pledged to accelerate innovation and speed access to treatments for rare diseases. That does not mean abandoning safety guardrails. It means applying those guardrails consistently and without unnecessary delay. Instead, the FDA broke written agreements, reversed approvals mid-process, and forced patient advocates to escalate their fight all the way to the White House.
The FDA’s attitude is illustrated by its handling of uniQure’s experimental treatment for Huntington’s disease — a rare, inherited disorder that progressively destroys nerve cells in the brain. First, the FDA reportedly agreed to let the manufacturer apply for approval. Then the agency changed its mind (in a move one former FDA senior official described as “evil”). Then it changed its mind again.
The particular drug or treatment is not the issue. Rather, this is a general problem: a notoriously bureaucratic agency is dead set on sticking to protocols that slow innovation in the United States and force healthcare firms to lose focus on the work we need them to do: curing diseases.
I work as a healthcare broker and consultant. Every day I sit across from clients negotiating things such as generic drug costs and off-label prescriptions — medicines so routine we barely register them as innovations anymore.
But many routine medicines began as experimental treatments for a single condition before proving useful for many others.
For example, Ibuprofen started as a prescription drug for rheumatoid arthritis. Gabapentin was initially approved for epilepsy; today, it is among the most prescribed drugs in the country, used off-label for nerve pain, anxiety, and alcohol withdrawal. Ozempic and other GLP-1 weight-loss drugs were a treatment for type 2 diabetes and are now helping millions of Americans lose weight.
None of that downstream medicine exists without the upstream approval. Nor were the prototypes sloppily or recklessly developed just because they were innovative. The molecular pathways mapped, the delivery mechanisms invented, and the safety profiles established for one disease do not disappear once a drug reaches market. They become the scaffolding on which the next generation of miracle cures is built.
This ripple effect isn’t unique to neurodegenerative diseases. A therapy known as exon-skipping used to treat a rare condition known as Duchenne Muscular Dystrophy — a progressive muscle-wasting condition that robs boys of mobility in childhood — works by bypassing defective genetic code so the body can behave normally.
These drugs are on the market under accelerated approval but face additional government demands that leave their future uncertain. The muscle wasting seen in DMD and age-related muscle loss — known as sarcopenia — shares enough biology that advances in one can lead to treatments for the other.
That is how American medical innovation has always worked. A breakthrough approved for a few thousand patients gets studied, refined, and eventually repurposed — first as an off-label treatment that courageous physicians discover works better for their patients, then as a generic that becomes standard of care for millions. The drug becomes a $12 bottle at the pharmacy, and nobody remembers it was once a risky, expensive bet on a rare disease.
“Risky” doesn’t have to mean risky for patients, though there will always be financial risks for innovators and entrepreneurs in healthcare. No one is calling for a “Wild West” that would entail wide-scale patient risk. The FDA is — and should be — a regulator, not a rubber stamp. But what rare disease families need, what every American needs, is reassurance that the FDA will act transparently, and with sensitivity to unique circumstances that don’t fit in the general mold.
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Bureaucrats always say they’re putting patients first, that we want to avoid drugs that could hurt people after they are approved. But no patient advocate wants their loved one harmed; they simply know that without treatment, the risk is 100% early death. They know we can keep all the necessary safety steps, and we can make them happen faster. Does anyone truly believe that the federal government’s drug approval process today is as streamlined and efficient as it possibly could be?
The recent reversal on Huntington’s disease is a sign that the agency can be pushed in the right direction. With new leadership taking shape, the FDA now has a chance to restore predictability, accountability, and trust. The question is whether it will seize that opportunity — or whether the next family will have to take their fight to Washington just to get the agency to honor its own word.
Matt Cover is a healthcare and employee benefits consultant and a former Washington, D.C. political journalist.