5 ms·

I think the FDA was in a difficult spot. There have been no new Alzheimers drugs in decades and patients are desperate for treatments. There is reason to believ

I think the FDA was in a difficult spot. There have been no new Alzheimers drugs in decades and patients are desperate for treatments. There is reason to believe that beta amyloid blockers need to be given in the earliest stages of Alzheimers (when diagnosis is difficult) to have any impact on cognitive decline. The trials did not focus on early stage patients, so there may be positive impacts if it can be given early enough.

compiler-guy 5y ago
Great! So let's figure out a way to make this study happen so that the drug can be given effectively (if it does indeed work) rather than have a blanket approval of a drug that lacks any proof of efficacy at all. That a drug hasn't been approved for a treatment in years is no reason to approve one that doesn't work.
- majormajor 5y ago
  > So let's figure out a way to make this study happen so that the drug can be given effectively (if it does indeed work) rather than have a blanket approval of a drug that lacks any proof of efficacy at all. It isn't a blanket approval, but it does sound way too generous in terms. From the linked article: > 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. > They will be absolutely overjoyed to do that, of course, because the whole time that’s going, they will be selling the first drug that (in theory) targets the etiology of Alzheimer’s. The backed-up demand is going to be gigantic, and Biogen is going to make enormous amounts of money. They have nine years, as it turns out, to get this trial done, and I feel safe in predicting that it’s going to take alllll niiiiine loooong sloooow years to get this done.
- jasonlaramburu 5y ago
  I guess the thinking is that it could take years to know whether it's working or not through an RCT. I believe they are currently allowed 9 years by FDA to show efficacy. If the side effects aren't significant why not let people in need try it?
  - robbiep 5y ago
    The side effects are significant. It comes with some risk of brain bleeds and a high risk of cerebral oedema in patients who have APOe4. This is an important genetic profile for those who understand Alzheimer’s, as homozygotes for APOe4 are 12x more likely to develop Alzheimer’s. This is a cop out decision by the FDA. Efficacy can be determined by a RCT - instead a decision was made which supports biogen’s share price
hmahncke 5y ago
The trial did focus on early stage patients - mild cognitive impairment (MCI) and early stage AD to be precise.

compiler-guy 5y ago

Great! So let's figure out a way to make this study happen so that the drug can be given effectively (if it does indeed work) rather than have a blanket approval of a drug that lacks any proof of efficacy at all. That a drug hasn't been approved for a treatment in years is no reason to approve one that doesn't work.

majormajor 5y ago
> So let's figure out a way to make this study happen so that the drug can be given effectively (if it does indeed work) rather than have a blanket approval of a drug that lacks any proof of efficacy at all. It isn't a blanket approval, but it does sound way too generous in terms. From the linked article: > 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. > They will be absolutely overjoyed to do that, of course, because the whole time that’s going, they will be selling the first drug that (in theory) targets the etiology of Alzheimer’s. The backed-up demand is going to be gigantic, and Biogen is going to make enormous amounts of money. They have nine years, as it turns out, to get this trial done, and I feel safe in predicting that it’s going to take alllll niiiiine loooong sloooow years to get this done.
jasonlaramburu 5y ago
I guess the thinking is that it could take years to know whether it's working or not through an RCT. I believe they are currently allowed 9 years by FDA to show efficacy. If the side effects aren't significant why not let people in need try it?
- robbiep 5y ago
  The side effects are significant. It comes with some risk of brain bleeds and a high risk of cerebral oedema in patients who have APOe4. This is an important genetic profile for those who understand Alzheimer’s, as homozygotes for APOe4 are 12x more likely to develop Alzheimer’s. This is a cop out decision by the FDA. Efficacy can be determined by a RCT - instead a decision was made which supports biogen’s share price

hmahncke 5y ago

The trial did focus on early stage patients - mild cognitive impairment (MCI) and early stage AD to be precise.