The context here (since the title isn't too revealing), is that the FDA expanded approval for a drug despite essentially unanimous didagreement from the scientists actually analyzing the drug. Derek Lowe, a well-respected pharma blogger, writes about how this was a big mistake and a very bad look for the FDA.
I'm seeing all sorts of negative takes on this. But I read one interesting take on reddit from a father of 2 kids with DMD that had a very specific and logical reason for supporting it (other than the treatment helped his kid).<p>Essentially the efficacy test is a 3 point scale that measures standing and walking. It's not granular enough to get real statistical significance on, and never will be unless another kind of test is defined. So the tests are basically always doomed to fail rigid scrutiny by the FDA.<p>In the meantime they're getting dramatically effective results, and Peter Marks is bucking the FDA's traditionally conservative stance to make the product available to the few who desperately need it. I'll eat my words if someone finds he was paid off for this. I think is a truly miraculous kind of therapy, and I hope to one day have regularly available gene therapy for my own afflictions.<p><a href="https://www.reddit.com/r/technology/comments/1dlsset/comment/l9rtb9q/" rel="nofollow">https://www.reddit.com/r/technology/comments/1dlsset/comment...</a>