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.