4 ms·

I get the qualms around this approval of a drug that very plausibly does not work, but I feel that too much ire is being directed at the FDA instead of at Medic

I get the qualms around this approval of a drug that very plausibly does not work, but I feel that too much ire is being directed at the FDA instead of at Medicare for paying for any approved drug. That's the core issue here — not that Biogen is allowed to sell the drug, but that public funds will be drained to pay for it. Yeah sure this might not work, but the FDA is reasonably confident it doesn't make things _worse_, and if it does actually work, there's immense potential for lives improved. Alzheimer's is truly a devastating blow to any family with a member who get it. So why not approve it? Bare minimum, we'll get more data.

jmcgough 5y ago
The data does not show that it works, and it was on a very specific sample (pre-alzheimers or early Alzheimer's). The drug costs $50k so they are profiting from desperate people. And yes, it can make things worse: "Most patients with AD (up to 75 percent) have a specific gene, called apolipoprotein E (APOE) ε4 allele; having this particular gene places a person at great risk of developing Alzheimer’s disease. Unfortunately, when AD patients with the APOE4 gene are given drugs like Aduhelm they are much more likely to develop brain edema and cerebral microhemorrhages. Indeed, in the BIOGEN trials, almost 50 percent of the APOE4 patients developed brain edema and hemorrhaging. In addition, these symptoms were accompanied by malignant hypertension and epileptic seizures."
shusaku 5y ago
Ironically usually the complaints go the other way, where people are mad a drug can’t be prescribed because the FDA isn’t convinced of it’s efficacy. But I’m not so sure that decoupling the decision is so easy. Patients get very upset if insurance doesn’t cover something…
tsimionescu 5y ago
> Yeah sure this might not work, but the FDA is reasonably confident it doesn't make things _worse_, and if it does actually work, there's immense potential for lives improved. Alzheimer's is truly a devastating blow to any family with a member who get it. So why not approve it? Bare minimum, we'll get more data. The reason is right there in what you're saying: the FDA is meant to protect people from predatory drug companies who know they are desperate to try anything to help their loved ones. If Biogen were selling homeopathic Alzheimer's cures, would you still think they should have been approved?
- evandijk70 5y ago
  Any data you will get from this approval will be very hard to interpret, as no randomization is done, so there is no proper control group. Moreover, proven safety is not sufficient to approve a drug. For experimental drugs without alternative treatment, it can be given as part of the compassionate use program. I think you could make an argument that the drug fits there, but approval sends the wrong message (ie. proven safety AND efficacy).
streptomycin 5y ago
Normal procedure for the FDA is to approve only effective drugs, not just drugs that are safe but might not actually work. If on a whim they've decided to change that, Medicare needs to be updated to be more skeptical of FDA-approved drugs. Unfortunately, our government is not very good at things like passing laws, so likely it will be quite some time before Medicare is adjusted. Very clever exploitation of an opportunity to basically steal billions of dollars from the public. I can't even be that mad, it's such an evil genius plan. Nicely done, Biogen.
- refurb 5y ago
  No. This is an intentional accelerated approval. Approved based “likelihood of clinical benefit based on bio marker changes”. The FDA is NOT saying it definitely works, it’s saying there is enough data to give early access.
  - lotsofpulp 5y ago
    Exactly. CMS is free to declare it insufficiently effective to be worth paying for.
    - refurb 5y ago
      CMS can do a “Coverage Determination” as they often do and either not cover it at all (unlikely) or severely limit it’s use (likely) potentially to patients who agree to be a part of a registry where outcomes are collected.
akvadrako 5y ago
Agreed. The FDA acting as a gatekeeper also keeps people from access to life saving therapies. It would be better if they allowed any drugs to be sold, but just labeled them as "safe" or "effective".

jmcgough 5y ago

The data does not show that it works, and it was on a very specific sample (pre-alzheimers or early Alzheimer's). The drug costs $50k so they are profiting from desperate people. And yes, it can make things worse: "Most patients with AD (up to 75 percent) have a specific gene, called apolipoprotein E (APOE) ε4 allele; having this particular gene places a person at great risk of developing Alzheimer’s disease. Unfortunately, when AD patients with the APOE4 gene are given drugs like Aduhelm they are much more likely to develop brain edema and cerebral microhemorrhages. Indeed, in the BIOGEN trials, almost 50 percent of the APOE4 patients developed brain edema and hemorrhaging. In addition, these symptoms were accompanied by malignant hypertension and epileptic seizures."

shusaku 5y ago

Ironically usually the complaints go the other way, where people are mad a drug can’t be prescribed because the FDA isn’t convinced of it’s efficacy. But I’m not so sure that decoupling the decision is so easy. Patients get very upset if insurance doesn’t cover something…

tsimionescu 5y ago

> Yeah sure this might not work, but the FDA is reasonably confident it doesn't make things _worse_, and if it does actually work, there's immense potential for lives improved. Alzheimer's is truly a devastating blow to any family with a member who get it. So why not approve it? Bare minimum, we'll get more data. The reason is right there in what you're saying: the FDA is meant to protect people from predatory drug companies who know they are desperate to try anything to help their loved ones. If Biogen were selling homeopathic Alzheimer's cures, would you still think they should have been approved?

evandijk70 5y ago
Any data you will get from this approval will be very hard to interpret, as no randomization is done, so there is no proper control group. Moreover, proven safety is not sufficient to approve a drug. For experimental drugs without alternative treatment, it can be given as part of the compassionate use program. I think you could make an argument that the drug fits there, but approval sends the wrong message (ie. proven safety AND efficacy).

streptomycin 5y ago

Normal procedure for the FDA is to approve only effective drugs, not just drugs that are safe but might not actually work. If on a whim they've decided to change that, Medicare needs to be updated to be more skeptical of FDA-approved drugs. Unfortunately, our government is not very good at things like passing laws, so likely it will be quite some time before Medicare is adjusted. Very clever exploitation of an opportunity to basically steal billions of dollars from the public. I can't even be that mad, it's such an evil genius plan. Nicely done, Biogen.

refurb 5y ago
No. This is an intentional accelerated approval. Approved based “likelihood of clinical benefit based on bio marker changes”. The FDA is NOT saying it definitely works, it’s saying there is enough data to give early access.
- lotsofpulp 5y ago
  Exactly. CMS is free to declare it insufficiently effective to be worth paying for.
  - refurb 5y ago
    CMS can do a “Coverage Determination” as they often do and either not cover it at all (unlikely) or severely limit it’s use (likely) potentially to patients who agree to be a part of a registry where outcomes are collected.

akvadrako 5y ago

Agreed. The FDA acting as a gatekeeper also keeps people from access to life saving therapies. It would be better if they allowed any drugs to be sold, but just labeled them as "safe" or "effective".