Know the questions a skeptical FDA reviewer will probe — before you're in the room.
In January 2026, the FDA released updated guidance on the use of Bayesian methods in clinical trials. The document does not read like a manifesto. It reads like an attempt to reconcile competing statistical cultures under real regulatory constraints. On its surface, it is pragmatic and flexible, welcoming Bayesian designs
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
