Know the questions a skeptical FDA reviewer will probe — before you're in the room.
When the Pfizer/BioNTech BNT162b2 trial reported 95% efficacy in November 2020, the world saw a scientific triumph. What most people missed, and what many statisticians still underappreciate, is that the trial's primary analysis was Bayesian. Not frequentist group sequential boundaries. Not O'Brien-Fleming. A posterior probability
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
