Three FDA Advisers Resign over Agency’s Approval of Alzheimer’s Drug

https://www.nytimes.com/2021/06/10/health/aduhelm-fda-resign-alzheimers.html

by nceqs3 • 5 years ago

76 40 5 years ago

nytimes.com

eganist• 5 years ago

What's stopping revocation of approval? Or is that fixed in stone now?

chews• 5 years ago

Stock prices mostly

phnofive2• 5 years ago

The manufacturer, Biogen, is ordered to continue studying the efficacy of the drug as a condition of approval. The results of this continued investigation are due in - seriously - nine years. Meanwhile, anyone with any stage the disease will receive treatment; fortunately (?), Medicare is expected to bear the burden of this expense.

ALittleLight2• 5 years ago

I really think the FDA approval system is backwards, especially for a terminal disease like Alzheimer's that we have no good treatment for. In my view we should encourage people to try whatever with a minimum of regulation and approval and then approve the best options for Medicare.

I imagine companies pitching (and discounting) any idea they can come up with and patients signing up for the best deals. As soon as someone starts to show something promising they get tax payer money and become locked in as the cure.

I just don't see any reason not to go balls to the wall on experimentation when you are treating the dying elderly. My grandfather died with some type of dementia. If I were in charge of his care I would have taken any experimental treatment over just letting him die with medical care that didn't have a chance of saving him. Better to take 1% chance of cure and 99% death than 100% chance of death.

Akinato2• 5 years ago

There's a lot of great arguments presented in the article for why we shouldn't go "balls to the wall on experimentation". The drug costs $56k a year, can cause some severe brain bleeding and swelling, and requires regular MRIs which can put quite a strain (and cost) on the system. These are some significant negatives for a drug that hasn't been proven to be effective at all.

mchusma2• 5 years ago

The drug only costs $56k because medicare can't negotiate. The parent was saying the bar for medicare paying for it should be decoupled from the bar for banning it. I agree.

ALittleLight2• 5 years ago

I think that makes the case for balls to the wall experimentation. Let whoever is interested and can afford it try this drug. If they have good results, lets cover it with medicaid. If not, try something else.

If we cut out regulations and trials and requirements and so on for what medical treatments people can try, then a bunch of stuff will be tried. Most of it will be bad, but some may show effect and we can iterate on that and get better medicines.

I don't advocate this style for every possible treatment. If we already have good treatments or if the illness isn't too bad, then we definitely should not risk things on trials. However, in this case, it is a disease that kills old people and we don't have any good treatments for it. Why not let people who want to experiment aggressively.

blendergeek• 5 years ago

Unfortunately, this sort of experimentation will simply lead to much quackery. As it stands, there is no cure for dementia. Allowing every company to market their own "treatment" does not make the situation better. It only allows the companies with the slickest marketing to exploit the elderly (and their families who desperately want to feel they did "all they could"). All the while, real dementia research (controlled, scientific research) may actually slow down as people leave clinical trials because they already bought some medicine.

nradov2• 5 years ago

Dementia is a symptom of multiple different conditions, not a disease per se. Sometimes the symptom can be cured by treating the root cause.

For example, an elderly woman I know was experiencing dementia. Her physicians ran a series of tests and diagnosed a bone marrow disorder. The dementia was caused by lack of oxygen to the brain from insufficient red blood cell production. So they cured the dementia by administering periodic blood transfusions.

mchusma2• 5 years ago

Right now it's worse than you state. Any completely unprovem acupuncture, herbal, chiropractic, homeopathic, etc treatment can get sold with basically no restrictions. Only medicine requires science. It's the worst of all worlds right now.

You also have to really struggle to convince me that there is an ethical case of banning someone from doing what they want with their own body.

I mean, you would probably say someone it is good to donate to medical research. You would also probably say it is good to volunteer for a medical study. But it's somehow wrong to give money to get the treatment? Makes no sense to me.

K0balt• 5 years ago

So we make it free until verified. Problem solved, filters for frauds, prolongs a few lives, ends pointless suffering in others, and creates a huge block of efficacy data. Win/win

hangonhn• 5 years ago

Sherwin Nuland wrote in his book "How We Die" that he really regretted getting experimental treatment for his brother who was dying of cancer. The side effects of the drugs were quite painful and his brother ended up dying anyways. He argued that people who are going to die should be allowed to die in the most peaceful and least painful way possible rather than gamble on some unproven treatment. That said, Alzheimer's is such a horrific disease that it's hard to imagine a peaceful or pain free death. People literally starve to death from "forgetting" the ability swallow. Sorry to hear about your loss. Nuland talked about euthanasia in his book as well. He gave an example of a professor who had Alzheimer's and decided to take his own life rather than wait for the disease to progress. Rather than forcing people like that to do that, Nuland believes palliative care and euthanasia has a role to play in such cases.

uncletammy2• 5 years ago

It seems like it would useful to have some kind of probationary approval for situations like this. If the data isn't convincing and the only alternative patients have is death, approve the drug under the condition that the manufacturer must greatly subsidize the cost until they have data that is convicing or the manufacturer loses confidence and withdraws from the market.

This might be a terrible idea given how complex and delicate the incentive structures are in healthcare.

nradov2• 5 years ago

That's what large scale Stage 3 clinical trials are supposed to be.

IfOnlyYouKnew• 5 years ago

That would remove all incentives to actually seek regulatory approval for drugs. We'd end up with an unlimited number of treatments trying to convince desperate laypeople of their benefits.

Another angle: yes, you would be willing to try any experimental drug you're reading about these days. So would I. But that's because the current regulatory system ensures that "experimental drugs" are usually at least good-faith efforts to produce a working, beneficial drug.

throwawayboise• 5 years ago

I don't think we should be wildly experimenting with cures for terminal illness in the elderly. I don't think we should be putting much effort into them at all, in fact. The elderly are going to die soon no matter what you do. We should be focusing our efforts on things that are terminal at a young age.

beachy• 5 years ago

You may think differently as you get older.

axiolite• 5 years ago

The age at which someone is elderly and likely to die soon is much higher than it used to be, thanks to the medical community NOT sharing your opinion and giving up on the elderly.

unishark• 5 years ago

This is basically how the law currently works.

If you are terminally ill you can apply for compassionate use from the FDA. And/or join a study. I think I read a while back that some large percentage of people dying of cancer were members of studies for drugs in process.

You can also get permission directly from a doctor under the "right to try" bill that was passed a few years ago.

I think all options must be after phase I is passed if I remember

resoluteteeth2• 5 years ago

This drug has already failed to be effective, it's extremely expensive, and it has bad side effects that require periodic MRIs to check for brain inflammation.

If the amyloid plaque hypothesis is wrong (which it almost certainly is based on the failure of other drugs) the destruction if amyloid plaques is likely to be harmful to the brain with no benefit.

Looking this as a 1% chance of a cure is not really accurate because if this drug was really capable of completely curing Alzheimer's there is no way the existing trials wouldn't have found that.

The article also describes additional reasons why it is generally bad to approve drugs that most likely don't actually work.

prepend• 5 years ago

This would be an ok mindset if the cost for treatment was zero or close to zero.

Currently, the cost of medication is very high (>$50k) and the vast majority is paid for by insurance, specifically Medicare.

So it’s a small chance for cure (maybe 1%) vs the cost that’s the equation.

I think this would incentivize lots of other drugs that are very expensive but known to not cure, or could potentially cure and the “science facade” would actually crowd out other potential cures.

Look at the supplement industry where there really isn’t any regulation, it’s mostly homeopathy taking advantage of people who want a fix. It’s like lotto in that it’s a waste of money but gives hope.

I frequently hear “just try it, what’s the harm” but the harm is the cost of trying lots of things. And a system where trying it incentivizes marketing and not cures isn’t very helpful.

uncletammy2• 5 years ago

https://archive.is/ztBWv

briefcomment• 5 years ago

Can someone who has access tell us why the price is so high? It seems cruel to make a medical treatment so expensive. You know some people will do anything to help loved ones overcome Alzheimer's, no matter the cost. If the high cost is avoidable, then it's predatory exploitation of inelastic demand.

peytn• 5 years ago

Likely years ago somebody at Biogen used a spreadsheet to calculate NPV under a range of scenarios, and from that decided to license this drug from Neurimmune. So it’s this or nothing, unfortunately.

bsder• 5 years ago

If it worked, $56K per year for a brand new drug for Alzheimer's isn't really that bad.

The problem is that is doesn't work. However, people will feel compelled to get it for their suffering relative as it is the only "approved" treatment.

mkhpalm• 5 years ago

Go read up on the history of ACTH (Acthar Gel) if you want to see how far this sort of stuff goes. Short summary is its a medication that cost approx $20 a vial for 50+ years and then got hiked to $40,000 a vial in the United States. It was the best known (gold standard) treatment for things like infantile spasms in newborns.

resoluteteeth2• 5 years ago

It's a monoclonal antibody. Biologics are expensive to produce.

phnofive2• 5 years ago

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• 5 years 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.

jolux3• 5 years 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.

spfzero• 5 years 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?

0xy• 5 years ago

The FDA approved the use of OxyContin, including banning the original formulation when the patent was about to expire so the Sacklers could change a small detail and continue to profit from the massacre it created. Why was this the straw that broke the camels back?

The FDA serves Big Pharma, not citizens.

Three FDA Advisers Resign over Agency’s Approval of Alzheimer’s Drug

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