Well, being a biostat just got more difficult. FDA, specifically the Boss in Charge Dr. Janet Woodcock, decided by fiat to approve Sarepta's eteplirsen, branded Exondys. The MoA data presented by the sponsor was not statistically different from 0, in aggregate, and no evidence that what dystrophin (the target compound) was produced was clinically meaningful. Woodcock threw not only the outside panel of experts, but also her staff under the bus. Both sets of experts saw eteplirsen for what it is: a (estimated) $400,000 saline solution. You wonder why healthcare cost goes nuts? This is one of the main reasons. The DMD parents, who lobbied ceaselessly for approval, win. Or so they think. When the boys die on schedule, they'll be really angry. That's not a good thing. If Exondys made some level of difference for all DMD patients, there might be some sense to this. But Exondys only affects 13% of DMD patients.
Sarepta is on the hook to conduct a confirmatory trial. I wouldn't hold my breath; they'll continue to find excuses, just as they've done so far.
It's a sad day for data.