5 ms·

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

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.

Akinato 5y 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.
- mchusma 5y 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.
  - ethanbond 5y ago
    Parent is saying very clearly that you should not need FDA approval to sell a drug that claims to treat some specific diseases.
- ALittleLight 5y 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.
  - ethanbond 5y ago
    One problem is that it’s actually extremely difficult to derive meaningful information from data collected outside of the context of a clinical trial. So you’d be giving greenlight to tons of fraudsters, exposing people to potentially way more suffering than their baseline disease causes, AND not learning anything from doing so.
    - Akinato 5y ago
      On top of that, we currently have a grossly overburdened healthcare system that in no way could support this. This presents so many clear problems that I wonder if the people are thinking critically about the long term effects of an unregulated drug market. 1. Knowledge -> How will doctors possibly keep track of the knowledge of all of these new drugs? If they don't need to be verified beyond a claim, they could be getting thousands of options. How do they pick between one drug with no proof, and another drug with no proof -- especially if they all have negative side effects? 2. Data -> All of the data for these drugs will be extremely if not impossible to derive. This will probably result in it taking even longer for evidence of the drug's efficacy to be built. 3. Resources -> Hospitals across North America are understaffed and overworked. How could they possibly find time to not only learn the entire treatment path for new drugs and all the necessary information, but to do this countless times across all their patients? MRI machines are already backlogged quite a bit, do we really think they could weather the storm of an unregulated drug market? 4. Liability -> Whether we like it or not, this adds quite a bit of liability to a hospital. Insurance will absolutely be against this, and would likely greatly increase the number of people trying to sue the hospital. 5. Fallout/Complications -> Can you imagine the strain of the additional complications that arise out of allowing free use of untested drugs? Imagine how much more expensive a patient would be if they got a brain bleed from a medication that they would never have had previously. This isn't just like "I tried a drug and it did nothing", it's more like "I tried a drug that had the potential to kill me. I had severe complications that required me to be in the hospital for 2 months but the drug still did nothing to help my problem." Effectively, we'd be breaking our own healthcare system by breaking down our regulations. Regulations are written in blood. They're there for a reason.
blendergeek 5y 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.
- nradov 5y 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.
- mchusma 5y 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.
  - jolux 5y ago
    Medical claims have a regulatory regime around them though, and alternative therapies are generally not allowed to make medical claims. This is not enforced as carefully as it should be in my opinion, but it definitely exists.
  - jleyank 5y ago
hangonhn 5y 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.
uncletammy 5y 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.
- nradov 5y ago
  That's what large scale Stage 3 clinical trials are supposed to be.
IfOnlyYouKnew 5y 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 5y 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.
- abraae 5y ago
  You may think differently as you get older.
- axiolite 5y 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 5y 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
resoluteteeth 5y 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 5y 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.

Akinato 5y 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.

mchusma 5y 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.
- ethanbond 5y ago
  Parent is saying very clearly that you should not need FDA approval to sell a drug that claims to treat some specific diseases.
ALittleLight 5y 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.
- ethanbond 5y ago
  One problem is that it’s actually extremely difficult to derive meaningful information from data collected outside of the context of a clinical trial. So you’d be giving greenlight to tons of fraudsters, exposing people to potentially way more suffering than their baseline disease causes, AND not learning anything from doing so.
  - Akinato 5y ago
    On top of that, we currently have a grossly overburdened healthcare system that in no way could support this. This presents so many clear problems that I wonder if the people are thinking critically about the long term effects of an unregulated drug market. 1. Knowledge -> How will doctors possibly keep track of the knowledge of all of these new drugs? If they don't need to be verified beyond a claim, they could be getting thousands of options. How do they pick between one drug with no proof, and another drug with no proof -- especially if they all have negative side effects? 2. Data -> All of the data for these drugs will be extremely if not impossible to derive. This will probably result in it taking even longer for evidence of the drug's efficacy to be built. 3. Resources -> Hospitals across North America are understaffed and overworked. How could they possibly find time to not only learn the entire treatment path for new drugs and all the necessary information, but to do this countless times across all their patients? MRI machines are already backlogged quite a bit, do we really think they could weather the storm of an unregulated drug market? 4. Liability -> Whether we like it or not, this adds quite a bit of liability to a hospital. Insurance will absolutely be against this, and would likely greatly increase the number of people trying to sue the hospital. 5. Fallout/Complications -> Can you imagine the strain of the additional complications that arise out of allowing free use of untested drugs? Imagine how much more expensive a patient would be if they got a brain bleed from a medication that they would never have had previously. This isn't just like "I tried a drug and it did nothing", it's more like "I tried a drug that had the potential to kill me. I had severe complications that required me to be in the hospital for 2 months but the drug still did nothing to help my problem." Effectively, we'd be breaking our own healthcare system by breaking down our regulations. Regulations are written in blood. They're there for a reason.

blendergeek 5y 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.

nradov 5y 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.
mchusma 5y 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.
- jolux 5y ago
  Medical claims have a regulatory regime around them though, and alternative therapies are generally not allowed to make medical claims. This is not enforced as carefully as it should be in my opinion, but it definitely exists.
- jleyank 5y ago

hangonhn 5y 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.

uncletammy 5y 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.

nradov 5y ago
That's what large scale Stage 3 clinical trials are supposed to be.

IfOnlyYouKnew 5y 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 5y 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.

abraae 5y ago
You may think differently as you get older.
axiolite 5y 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 5y 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

resoluteteeth 5y 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 5y 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.