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

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.

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.

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

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

Stock prices mostly

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.