[T]he patient advocacy groups took victory laps after Relyvrio was approved, and they were a big part of the pressure that made the FDA reverse its initial correct decision.
Motivated reasoning, whether by commercial interests, patient advocates, or any other group, is inimical with truth-finding.
Though on the grounds of potential benefit vs. known harm (which is assessed in Phase I/II trials) and partial knowledge, a provisional approval in the case of a treatment for a chronic, progressive, and fatal condition is reasonably defensible. The FDA's conditional approval was based on Phase III trials showing efficacy. The dice were rolled, though the gamble in this case proved fruitless.
In this case, the FDA had approved the drug, though conditionally.
Second 'graph of TFA:
A second advisory committee meeting was convened, and if that phrase sounds odd to you, it should. That's a very unusual thing to do. This one voted for approval, and the FDA did approve the drug in September of 2022. There was a condition, though: Amylyx was already working on a Phase III trial, and they committed to withdrawing the drug if this trial showed no efficacy.
Why would you test something for efficacy before you're sure it's safe? You'll end up testing a bunch of harmful substances that don't actually do anything.
Maybe because the real standard is "the benefits out weigh the risks" you might want a drug that has a 10% chance of harm if it has a 90% chance of benefit, for a condition that is terminal if untreated.
[T]he patient advocacy groups took victory laps after Relyvrio was approved, and they were a big part of the pressure that made the FDA reverse its initial correct decision.
Motivated reasoning, whether by commercial interests, patient advocates, or any other group, is inimical with truth-finding.
Though on the grounds of potential benefit vs. known harm (which is assessed in Phase I/II trials) and partial knowledge, a provisional approval in the case of a treatment for a chronic, progressive, and fatal condition is reasonably defensible. The FDA's conditional approval was based on Phase III trials showing efficacy. The dice were rolled, though the gamble in this case proved fruitless.