Know the questions a skeptical FDA reviewer will probe — before you're in the room.
In 2019, FDA granted accelerated approval to voxelotor for sickle cell disease based on a surrogate endpoint: hemoglobin increase. The mechanism was sound. Voxelotor increased hemoglobin oxygen affinity, which should reduce sickling and improve outcomes. The biology made sense. The FDA agreed. Patients got access. In September 2024, the drug
Most statistical frameworks treat errors symmetrically. A false positive is bad. A false negative is bad. Control one, tolerate the other, and let the math do the rest. Clinical reality is not that tidy. Approving an ineffective therapy and withholding a potentially effective one are both errors, but they do
In September 2016, the FDA approved eteplirsen for Duchenne muscular dystrophy. The advisory committee had voted 7 to 6 against accelerated approval. The FDA's own review team recommended against it. The clinical program consisted of 12 boys, and western blot analysis showed a dystrophin increase of 0.93%
