As the world knows, the FDA approved Biogen’s anti-amyloid antibody today, surely the first marketed drug whose Phase III trial was stopped for futility. I think this is one of the worst FDA decisions I have ever seen, because – like the advisory committee that reviewed the application, and like the FDA’s own statisticians – I don’t believe that Biogen really demonstrated efficacy. No problem apparently. The agency seems to have approved it based on its demonstrated ability to clear beta-amyloid, and is asking Biogen to run a confirmatory trial to show efficacy.
So the FDA has, for expediency’s sake, bought into the amyloid hypothesis although every single attempt to translate that into a beneficial clinical effect has failed. I really, really don’t like the precedent that this sets: what doesn’t get approved, now? I suppose only things that definitely cause harm, because otherwise why not just ask for the same deal that Biogen got, and go out and prove efficacy while you turn a profit? I know that this is just the libertarian turn that many people have wished for, but I’m still not sanguine about it.