Know the questions a skeptical FDA reviewer will probe — before you're in the room.
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%
The Bayesian vs. frequentist debate usually centers on philosophy. Priors are subjective! No, they formalize existing knowledge! You're smuggling in assumptions! You're ignoring relevant information! It's a fun argument. It's also the wrong one, at least for regulatory decision-making. The real value
