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Yikes, read through to the end to understand why the FDA approved this drug despite no support (ten against, one uncertain) from their advisory panel: > Dr. St

Yikes, read through to the end to understand why the FDA approved this drug despite no support (ten against, one uncertain) from their advisory panel: > Dr. Stein also defended the agency’s decision to approve the drug for such a broad group of patients, saying it could be relevant beyond the early stages of Alzheimer’s. "Because amyloid is a hallmark of the disease through its entire course, the expectation is that this drug will provide benefit across that spectrum," Dr. Stein said. That expectation was not borne out as clinically significant in the trials, yet the beta-amyloid hypothesis lives on.

qzw 5y ago
What the FDA is doing is in effect allowing Biogen to run a large scale clinical trial while charging full price for the drug. This would normally be a terrible idea, but Alzheimer's is such a devastating and long lasting disease that patients as well as insurance companies might well be willing to subsidize Biogen just to find out if this thing really works or not. Such is the gloomy reality of an incurable, progressive, and fatal disease.
- jolux 5y ago
  This is a potentially dangerous direction to be taking the approval process in, though. It’s not meant to be for experimental treatments with at best inconclusive efficacy. Being an approved treatment is supposed to mean that an agent is efficacious against a disease.
  - dragonwriter 5y ago
    “Efficacious”, though shouldn't be viewed as a binary, but as a sliding scale where the cutoff for any particular application depends on the balance of risks, available alternative treatments, etc.
    - jolux 5y ago
      Why shouldn’t it be viewed as a binary? They couldn’t find any evidence it was effective! You can’t get much more of a zero than that. Out of eleven, ten members of the advisory board were against approval and one was uncertain. I’ve got no idea why this drug was approved, unless it was an emotional appeal to the plight of Alzheimer’s patients, which should not guide which treatments we use.
- spfzero 5y ago
  The problem here is that by “approving” the drug, the FDA implies that it truly works. People will take it expecting improvement, but their time will be wasted and their hopes dashed. The approval process itself will lose credibility, and that will hurt everyone. By being devastating and long lasting, Alzheimer’s is not exceptional, compared to say cancer or even CVD. This decision really changes the perception of what the FDA is there for, if not to ensure efficacy and safety, then what?

qzw 5y ago

What the FDA is doing is in effect allowing Biogen to run a large scale clinical trial while charging full price for the drug. This would normally be a terrible idea, but Alzheimer's is such a devastating and long lasting disease that patients as well as insurance companies might well be willing to subsidize Biogen just to find out if this thing really works or not. Such is the gloomy reality of an incurable, progressive, and fatal disease.

jolux 5y ago
This is a potentially dangerous direction to be taking the approval process in, though. It’s not meant to be for experimental treatments with at best inconclusive efficacy. Being an approved treatment is supposed to mean that an agent is efficacious against a disease.
- dragonwriter 5y ago
  “Efficacious”, though shouldn't be viewed as a binary, but as a sliding scale where the cutoff for any particular application depends on the balance of risks, available alternative treatments, etc.
  - jolux 5y ago
    Why shouldn’t it be viewed as a binary? They couldn’t find any evidence it was effective! You can’t get much more of a zero than that. Out of eleven, ten members of the advisory board were against approval and one was uncertain. I’ve got no idea why this drug was approved, unless it was an emotional appeal to the plight of Alzheimer’s patients, which should not guide which treatments we use.
spfzero 5y ago
The problem here is that by “approving” the drug, the FDA implies that it truly works. People will take it expecting improvement, but their time will be wasted and their hopes dashed. The approval process itself will lose credibility, and that will hurt everyone. By being devastating and long lasting, Alzheimer’s is not exceptional, compared to say cancer or even CVD. This decision really changes the perception of what the FDA is there for, if not to ensure efficacy and safety, then what?