Picture a parent whose child has Hunter syndrome. The disease attacks the body slowly, stealing movement and memory. There are 3 drugs in development that might help. The FDA has blocked or delayed all 3. On February 23, 2026, the same week the FDA released a new framework meant to speed up rare disease approvals, advocates staged a funeral outside the agency's doors. That image matters.
My read: the FDA is not wildly too strict, but it is definitely too slow, and its inconsistency is doing real harm.
The New Framework Sounds Better Than It Is
The FDA's new "Plausible Mechanism Framework" lets drug sponsors skip randomized controlled trials when patient populations are tiny. Instead, companies can use disease biology data, biomarkers, and natural history comparisons to show their drug works. That is a genuine improvement for a field where running a 500-person trial is sometimes impossible because only 300 people on earth have the condition.
Here is the catch: the FDA insists approval standards are "unchanged." Companies still need to show "substantial evidence of effectiveness." The framework just describes a different path to that same bar. Whether that path is actually accessible depends entirely on how FDA reviewers apply it case by case. And the Hunter syndrome decisions suggest they are not applying it generously.
To be fair to the FDA: the HHS response to the funeral protest made a reasonable point. Drugs approved on weak data can hurt people, especially children. The thalidomide era was not that long ago. Caution has earned its place in this building. But caution that becomes paralysis is its own kind of harm.
What Consistency Would Actually Look Like
Congress reauthorized the Rare Pediatric Disease Priority Review Voucher program through September 2029 as part of the Consolidated Appropriations Act, 2026. On March 13, 2026, the FDA used that voucher mechanism to approve EYLEA HD for a rare pediatric eye condition. So the tools for faster action exist. The FDA just uses them selectively.
The agency also approved ZYCUBO for Menkes disease earlier this year, its first rare disease approval of 2026. So it can say yes. The question is whether it is saying yes and no by coherent standards or by something closer to gut feel and internal politics. Right now, families with children who have Hunter syndrome are watching ZYCUBO get approved while their kids keep getting sicker. That inconsistency is what erodes trust.
What should change: the FDA needs to publish clear, condition-specific criteria for when biomarkers are sufficient as endpoints. Not a general framework. Specific guidance that tells a drug company: for Hunter syndrome, here is exactly what we will and will not count as evidence. That moves the debate out of the protest line and into the lab, where it belongs.
The agency also needs a faster appeals track for decisions that advocates challenge on scientific grounds. Right now, a denial can stall a drug for years while a family's window closes. A 90-day formal review by an independent scientific panel would give the FDA's decisions more credibility, not less.
The parent outside the FDA building in February was not asking the agency to approve a dangerous drug. They were asking it to explain, clearly, what evidence would actually change the answer. That is a reasonable ask. The FDA should be able to answer it.
