When it comes to reviewing drugs, the FDA’s job seems straightforward: make sure a drug is safe. Then make sure that it actually works and does what it is supposed to. It’s an essential mission that this deeply dysfunctional organization appears to be having trouble fulfilling. The EpiPen pricing scandal and the controversial approvals of flibanserin (Addyi) to improve underactive sexual desire in women and eteplirsen (Exondys 51) for treating Duchenne muscular dystrophy illustrate that the FDA appears to be having trouble following its own guidelines.
I have been working in investigational drug development for almost two decades. As a politically conservative medical scientist at the FDA — a supreme rarity, I assure you — who has worked as a medical officer and senior medical analyst, and as a former investigational medicine research scientist at Pfizer, I haven’t always agreed with regulatory decisions made by the FDA.