7 ms·

My take on this is counter-consensus. I don't believe that screening drugs based on effectiveness is a proper role for any government agency. There is a role fo

My take on this is counter-consensus. I don't believe that screening drugs based on effectiveness is a proper role for any government agency. There is a role for them to guard against fraud and to protect rights, but not to prevent people from making risky choices. Adults should not be protected from the decision to take any drug, however harmful, even if instantly fatal, if fraud or force is not involved. These are extraordinarily complex choices that seldom have a right answer for all possible patients. Therefore they should not be made centrally for everyone. We will learn more and faster from decentralized decision makers, that is, from massively parallel computation from a mass population of decision makers. Better would be for the only role of the FDA in decisions like this to be determining if the manufacturer is making fraudulent claims. Even better (because of greater computational parallelism) would be for this to be legally decided only by individual states. Best would be to treat adults as adults, and let them and their medical providers make such choices.

nabla9 5y ago
Your argument misses some of the important reasons of the approval process. Firstly, approval limits the legal liability for drugmakers. If we adopt a policy where approval does not exist. As long as the government takes part in the risk so it should have a say in the matter. > treat adults as adults, and let them and their medical providers make such choices. Signing out of medicare and all government assistance in case of becoming invalid, so that government is not in the hook for personal decisions would be a huge ask. If 10 million individuals make the same decision independently, and it backfires, that has negative consequences for the rest of the country. One decision does not matter, millions of decisions become collective matter.
kesselvon 5y ago
What a terrible idea. Nobody has the knowledge base to make a real informed decision on the efficacy of a drug. Not filtering for effectiveness is to endorse fraud and blame it on consumers when it happens. The whole reason the FDA is set up this way was due to the rampant fraud that was occurring before there were standards for what could be sold to consumers as medicine.
- yanderekko 5y ago
  >Nobody has the knowledge base to make a real informed decision on the efficacy of a drug. Informed decisions are not risk-free decisions. If you're saying that literally no one can provide informed consent to take a drug until years and years of trials have been performed, and their inability to provide informed consent invariably subjects them to functional degradation or death, then this is a perverse system, to say the least. Note that many people made the same arguments against fast-tracking COVID vaccines for public distribution, which undoubtedly saved thousands if not millions of lives.
  - disgruntledphd2 5y ago
    The FDA was required to approve all new drugs because of this: https://en.wikipedia.org/wiki/Thalidomide https://en.wikipedia.org/wiki/Thalidomide I personally agree that the FDA isn't always perfect (they should never have approved SSRI's, for instance) but claiming that there's no tradeoff here is just incorrect.
    - JoshuaDavid 5y ago
      Yes, and the TSA is required to check the shoes of all passengers who want to fly because of this: https://en.wikipedia.org/wiki/Richard_Reid https://en.wikipedia.org/wiki/Richard_Reid The fact the people who make a policy have a specific thing they want to achieve with that policy does not automatically make it a good policy. That remains true even if the thing they want to prevent is bad and could kill people.
      - disgruntledphd2 5y ago
        I met one of the thalidomide babies once, he was a PI for a research project I worked on. He was a relatively tall man, and had tiny arms that would have looked fine on a 2 year old. Stopping stuff like that seems like a reasonable thing for the FDA to do, even at the cost of it taking longer to approve drugs.
deschutes 5y ago
Efficacy and safety are intertwined. Safety can sometimes only be evaluated in the context of efficacy. For example, you wouldn't amputate a healthy arm. But for a gangrenous one, it may be the only option. For establishing efficacy and safety, trials are the only process that works. Nobody can predict the effects of drugs because the current understanding of biology is very incomplete. Thalidomide [1] shows the wisdom of the current approach. [1]: https://en.m.wikipedia.org/wiki/Thalidomide https://en.m.wikipedia.org/wiki/Thalidomide
Angostura 5y ago
> These are extraordinarily complex choices that seldom have a right answer for all possible patients. I think "is there any evidence that this drug actually works" can a remarkably simple decision, given the results of clinical trials. The fact that desperate people may be willing to blow tens of thousands of dollars on something that doesn't work, doesn't make it more complex.
tboyd47 5y ago
So, do you think people should be allowed to take ivermectin and "that drug" (the one that rhymes with mydoxyborofin) for COVID-19 if they want to, given that both of those have been studied for years and are known to have very little side effects?
- swader999 5y ago
  No, the patents on these have expired and nobody will make any money if we promote them. Also it would have been near impossible to get emergency use authorisation for these vaccines if we had at all listened to the science on these.
- thehappypm 5y ago
  You won't even say the name of the drug hydroxychloroquine? Trump really did a number on you!
  - tboyd47 5y ago
    head explodes
qudat 5y ago
I agree. The FDA should be a watch dog group that provides us with the knowledge to make our own decisions. How many people have died because a drug took too long or never made it to get approved?
dcolkitt 5y ago
I agree. And regardless of where you stand on the issue, the current regulations with regards to off-label usage are totally inconsistent. Doctors are perfectly free to prescribe drugs for conditions that are completely unrelated to the what they were approved for. Drug X is proven safe but not proven effective. It's illegal to use to treat condition Q under any circumstances. Drug Y is proven safe, proven effective for condition M. It's perfectly legal to use for unrelated condition Q. How does this make any sense?
- spiorf 5y ago
  Because it cuts economic incentive to produce ineffective (but safe) drugs on purpose.
  - SpicyLemonZest 5y ago
    Maybe, but it’s not clear to me that the FDA is (or should be) experts in weighing economic incentives for good behavior. There’s plenty of bad economic incentives created by the current FDA process; just ask the Lovaza guys, charging a 10x markup for fish oil. In an ideal world, I don’t think the FDA should have any role in drug approvals other than preventing direct medical harm.
    - deleted 5y ago
      [deleted]
radus 5y ago
This would likely lead to a pharmaceutical landscape that is more like the supplements industry -- no thank you.
alisonkisk 5y ago
That's not really relevant here. Safe drugs can be sold OTC with all the homeopathics. FDA approval here is about fraudulent claims of effectiveness. You argue that unsafe drugs should be available for sale too, but that's not what happened here and IMO it ignores the societal risks of unsafe things floating around were they can harm non-consenting people, as well as the rights of people who want to be free from transient temptation to make irrevocable decisions.
devoutsalsa 5y ago
I am a smart person, and I am not remotely qualified to make decisions about the risks associated with an untested drug. I’d like to know when I get a prescription that someone else went through the process of being a willing volunteer in a clinical trial.
- bencoder 5y ago
  Your doctor should be able to help with that and provide you only drugs that have completed clinical trials to your desired standard.
  - kesselvon 5y ago
    Doctors heavily rely on FDA to filter for effective drugs, they're not researchers with the skills to identify whether a drug has efficacy.
  - chinathrow 5y ago
    Your doctor is a smart person too but they can't simply check every single medication single handily. What even is "your desired standard" here?
    - greeneggs 5y ago
      Sure, but the doctor doesn't have to do much research in this case. This is front-page news in the NY Times. And about "every single medication": this is the only approved medication to delay clinical decline due to Alzheimer's. And there are only a few medications to treat symptoms. I know this isn't a perfect world, but it doesn't seem to be asking a lot for a doctor to know this. (Especially for a drug that currently costs $56K/year and presumably will cost more next year.)
      - setpatchaddress 5y ago
        You seem to have an incredibly optimistic view of the both the medical profession and human attention span.
burkaman 5y ago
Isn't it fraud to sell an Alzheimer's drug that doesn't prevent Alzheimer's?
- readams 5y ago
  Only if you make untrue claims. If you claim only what is proven, then it is not fraud.
  - burkaman 5y ago
    > ADUHELM is an amyloid beta-directed antibody indicated for the treatment of Alzheimer’s disease. - https://www.biogencdn.com/us/aduhelm-pi.pdf https://www.biogencdn.com/us/aduhelm-pi.pdf I believe the advisory panel's position is that it is not indicated for the treatment of Alzheimer's and that Biogen should not be allowed to say this. In their press release (https://investors.biogen.com/news-releases/news-release-details/fda-grants-accelerated-approval-aduhelmtm-first-and-only https://investors.biogen.com/news-releases/news-release-deta...), they explicitly describe it as an "Alzheimer's disease treatment". Again, I think the advisory panel considers this untrue, and certainly unproven. The FDA could have said "this drug is safe and may be sold, but you may not describe it as an Alzheimer's treatment, and you must explicitly state that it has been found to be ineffective as an Alzheimer's treatment in clinical trials".
  - danso 5y ago
    So how is Biogen going to get doctors to prescribe and people to pay $50K for a drug with no brand recognition nor eligibility for insurance, and no claims of actual effectiveness?
usefulcat 5y ago
The problem is that it costs $56k/year. Most people don't have that kind of money, so for the most part we're talking about whether that cost should be socialized, either via insurance or the government. That's an awful lot of money that could be used to treat a lot of other sick people. So it's not primarily a question of whether people should be allowed to take risks, it's more a question of how best to allocate finite resources.
- barsonme 5y ago
  Sure, but that is also not a question FFA should be answering.
jartelt 5y ago
None of this vision would work for a variety of reasons, but the biggest one is that insurance companies are not going to agree to pay for medications that are not proven effective. Your personal freedom to try whichever medications you want would be limited unless you are willing to pay out of pocket for everything.
galimaufry 5y ago
As I understand it the issue is * Currently the US welfare agencies will provide ~any FDA-approved drug for someone with the illness for which the drug was approved * In this case, they probably won't - in the worst case scenario it would double their spending on prescription drugs and seems unlikely to help anyone * So this is going to sever the link between FDA approval and medicaid spending. * Now you have an awkward situation where agencies that not really equipped to make judgements on dug effectiveness, and which are not insulated from politics, have to make those decisions. This could all work out for the best in the long term but may also be very awkward in the short term.
Clubber 5y ago
Wouldn't charging $56k / year for a drug that not only doesn't work, but has a high chance to induce brain swelling be considered fraud?
danso 5y ago
> I don't believe that screening drugs based on effectiveness is a proper role for any government agency...Better would be for the only role of the FDA in decisions like this to be determining if the manufacturer is making fraudulent claims. I think you come in with wrong assumptions. The FDA has little to no authority when it comes to off-label uses — doctors have a lot of leeway to prescribe drugs for whatever non-approved use they judge to be effective. So the FDA's role already is intertwined with marketing. A doctor can today prescribe Viagra as a treatment to Alzheimers, but it would be illegal for Pfizer to market Viagra for treating Alzheimers until it gets FDA approval for that specific use.
jcranmer 5y ago
> Better would be for the only role of the FDA in decisions like this to be determining if the manufacturer is making fraudulent claims. Let us recall some of the key facts about the drug in question here. There were two trials. One of them showed such little benefit that it was halted midtrial on the basis that it would be unethical to continue giving people a drug that didn't work. The second showed marginal benefit. Biogen argued that there is a rational explanation that explains the two trials that points to the drug working. The expert committee at the FDA had 10 people vote "that's bunk", 1 person vote "unsure", and nobody vote "I agree." That's a pretty strong endorsement for saying "Aduhelm treats Alzheimer's" is a fraudulent claim, and yet the FDA decided that the claim is merely disputed. The only way you can look on the FDA's decision favorably is if you know nothing about how drug development works, which is unfortunately true of almost the entire public. (Here's two facts that are important to keep in mind: most drug candidates don't work, and placebo effects are very real and surprisingly powerful).
mcguire 5y ago
I suppose there is some fine ethical reasoning that would distinguish between selling something that you know not to work and selling something that you don't know won't work. Of course, financially incentivizing ignorance is probably not the best precedent.

nabla9 5y ago

Your argument misses some of the important reasons of the approval process. Firstly, approval limits the legal liability for drugmakers. If we adopt a policy where approval does not exist. As long as the government takes part in the risk so it should have a say in the matter. > treat adults as adults, and let them and their medical providers make such choices. Signing out of medicare and all government assistance in case of becoming invalid, so that government is not in the hook for personal decisions would be a huge ask. If 10 million individuals make the same decision independently, and it backfires, that has negative consequences for the rest of the country. One decision does not matter, millions of decisions become collective matter.

kesselvon 5y ago

What a terrible idea. Nobody has the knowledge base to make a real informed decision on the efficacy of a drug. Not filtering for effectiveness is to endorse fraud and blame it on consumers when it happens. The whole reason the FDA is set up this way was due to the rampant fraud that was occurring before there were standards for what could be sold to consumers as medicine.

yanderekko 5y ago
>Nobody has the knowledge base to make a real informed decision on the efficacy of a drug. Informed decisions are not risk-free decisions. If you're saying that literally no one can provide informed consent to take a drug until years and years of trials have been performed, and their inability to provide informed consent invariably subjects them to functional degradation or death, then this is a perverse system, to say the least. Note that many people made the same arguments against fast-tracking COVID vaccines for public distribution, which undoubtedly saved thousands if not millions of lives.
- disgruntledphd2 5y ago
  The FDA was required to approve all new drugs because of this: https://en.wikipedia.org/wiki/Thalidomide https://en.wikipedia.org/wiki/Thalidomide I personally agree that the FDA isn't always perfect (they should never have approved SSRI's, for instance) but claiming that there's no tradeoff here is just incorrect.
  - JoshuaDavid 5y ago
    Yes, and the TSA is required to check the shoes of all passengers who want to fly because of this: https://en.wikipedia.org/wiki/Richard_Reid https://en.wikipedia.org/wiki/Richard_Reid The fact the people who make a policy have a specific thing they want to achieve with that policy does not automatically make it a good policy. That remains true even if the thing they want to prevent is bad and could kill people.
    - disgruntledphd2 5y ago
      I met one of the thalidomide babies once, he was a PI for a research project I worked on. He was a relatively tall man, and had tiny arms that would have looked fine on a 2 year old. Stopping stuff like that seems like a reasonable thing for the FDA to do, even at the cost of it taking longer to approve drugs.

deschutes 5y ago

Efficacy and safety are intertwined. Safety can sometimes only be evaluated in the context of efficacy. For example, you wouldn't amputate a healthy arm. But for a gangrenous one, it may be the only option. For establishing efficacy and safety, trials are the only process that works. Nobody can predict the effects of drugs because the current understanding of biology is very incomplete. Thalidomide [1] shows the wisdom of the current approach. [1]: https://en.m.wikipedia.org/wiki/Thalidomide https://en.m.wikipedia.org/wiki/Thalidomide

Angostura 5y ago

> These are extraordinarily complex choices that seldom have a right answer for all possible patients. I think "is there any evidence that this drug actually works" can a remarkably simple decision, given the results of clinical trials. The fact that desperate people may be willing to blow tens of thousands of dollars on something that doesn't work, doesn't make it more complex.

tboyd47 5y ago

So, do you think people should be allowed to take ivermectin and "that drug" (the one that rhymes with mydoxyborofin) for COVID-19 if they want to, given that both of those have been studied for years and are known to have very little side effects?

swader999 5y ago
No, the patents on these have expired and nobody will make any money if we promote them. Also it would have been near impossible to get emergency use authorisation for these vaccines if we had at all listened to the science on these.
thehappypm 5y ago
You won't even say the name of the drug hydroxychloroquine? Trump really did a number on you!
- tboyd47 5y ago
  head explodes

qudat 5y ago

I agree. The FDA should be a watch dog group that provides us with the knowledge to make our own decisions. How many people have died because a drug took too long or never made it to get approved?

dcolkitt 5y ago

I agree. And regardless of where you stand on the issue, the current regulations with regards to off-label usage are totally inconsistent. Doctors are perfectly free to prescribe drugs for conditions that are completely unrelated to the what they were approved for. Drug X is proven safe but not proven effective. It's illegal to use to treat condition Q under any circumstances. Drug Y is proven safe, proven effective for condition M. It's perfectly legal to use for unrelated condition Q. How does this make any sense?

spiorf 5y ago
Because it cuts economic incentive to produce ineffective (but safe) drugs on purpose.
- SpicyLemonZest 5y ago
  Maybe, but it’s not clear to me that the FDA is (or should be) experts in weighing economic incentives for good behavior. There’s plenty of bad economic incentives created by the current FDA process; just ask the Lovaza guys, charging a 10x markup for fish oil. In an ideal world, I don’t think the FDA should have any role in drug approvals other than preventing direct medical harm.
  - deleted 5y ago
    [deleted]

radus 5y ago

This would likely lead to a pharmaceutical landscape that is more like the supplements industry -- no thank you.

alisonkisk 5y ago

That's not really relevant here. Safe drugs can be sold OTC with all the homeopathics. FDA approval here is about fraudulent claims of effectiveness. You argue that unsafe drugs should be available for sale too, but that's not what happened here and IMO it ignores the societal risks of unsafe things floating around were they can harm non-consenting people, as well as the rights of people who want to be free from transient temptation to make irrevocable decisions.

devoutsalsa 5y ago

I am a smart person, and I am not remotely qualified to make decisions about the risks associated with an untested drug. I’d like to know when I get a prescription that someone else went through the process of being a willing volunteer in a clinical trial.

bencoder 5y ago
Your doctor should be able to help with that and provide you only drugs that have completed clinical trials to your desired standard.
- kesselvon 5y ago
  Doctors heavily rely on FDA to filter for effective drugs, they're not researchers with the skills to identify whether a drug has efficacy.
- chinathrow 5y ago
  Your doctor is a smart person too but they can't simply check every single medication single handily. What even is "your desired standard" here?
  - greeneggs 5y ago
    Sure, but the doctor doesn't have to do much research in this case. This is front-page news in the NY Times. And about "every single medication": this is the only approved medication to delay clinical decline due to Alzheimer's. And there are only a few medications to treat symptoms. I know this isn't a perfect world, but it doesn't seem to be asking a lot for a doctor to know this. (Especially for a drug that currently costs $56K/year and presumably will cost more next year.)
    - setpatchaddress 5y ago
      You seem to have an incredibly optimistic view of the both the medical profession and human attention span.

burkaman 5y ago

Isn't it fraud to sell an Alzheimer's drug that doesn't prevent Alzheimer's?

readams 5y ago
Only if you make untrue claims. If you claim only what is proven, then it is not fraud.
- burkaman 5y ago
  > ADUHELM is an amyloid beta-directed antibody indicated for the treatment of Alzheimer’s disease. - https://www.biogencdn.com/us/aduhelm-pi.pdf https://www.biogencdn.com/us/aduhelm-pi.pdf I believe the advisory panel's position is that it is not indicated for the treatment of Alzheimer's and that Biogen should not be allowed to say this. In their press release (https://investors.biogen.com/news-releases/news-release-details/fda-grants-accelerated-approval-aduhelmtm-first-and-only https://investors.biogen.com/news-releases/news-release-deta...), they explicitly describe it as an "Alzheimer's disease treatment". Again, I think the advisory panel considers this untrue, and certainly unproven. The FDA could have said "this drug is safe and may be sold, but you may not describe it as an Alzheimer's treatment, and you must explicitly state that it has been found to be ineffective as an Alzheimer's treatment in clinical trials".
- danso 5y ago
  So how is Biogen going to get doctors to prescribe and people to pay $50K for a drug with no brand recognition nor eligibility for insurance, and no claims of actual effectiveness?

usefulcat 5y ago

The problem is that it costs $56k/year. Most people don't have that kind of money, so for the most part we're talking about whether that cost should be socialized, either via insurance or the government. That's an awful lot of money that could be used to treat a lot of other sick people. So it's not primarily a question of whether people should be allowed to take risks, it's more a question of how best to allocate finite resources.

barsonme 5y ago
Sure, but that is also not a question FFA should be answering.

jartelt 5y ago

None of this vision would work for a variety of reasons, but the biggest one is that insurance companies are not going to agree to pay for medications that are not proven effective. Your personal freedom to try whichever medications you want would be limited unless you are willing to pay out of pocket for everything.

galimaufry 5y ago

As I understand it the issue is * Currently the US welfare agencies will provide ~any FDA-approved drug for someone with the illness for which the drug was approved * In this case, they probably won't - in the worst case scenario it would double their spending on prescription drugs and seems unlikely to help anyone * So this is going to sever the link between FDA approval and medicaid spending. * Now you have an awkward situation where agencies that not really equipped to make judgements on dug effectiveness, and which are not insulated from politics, have to make those decisions. This could all work out for the best in the long term but may also be very awkward in the short term.

Clubber 5y ago

Wouldn't charging $56k / year for a drug that not only doesn't work, but has a high chance to induce brain swelling be considered fraud?

danso 5y ago

> I don't believe that screening drugs based on effectiveness is a proper role for any government agency...Better would be for the only role of the FDA in decisions like this to be determining if the manufacturer is making fraudulent claims. I think you come in with wrong assumptions. The FDA has little to no authority when it comes to off-label uses — doctors have a lot of leeway to prescribe drugs for whatever non-approved use they judge to be effective. So the FDA's role already is intertwined with marketing. A doctor can today prescribe Viagra as a treatment to Alzheimers, but it would be illegal for Pfizer to market Viagra for treating Alzheimers until it gets FDA approval for that specific use.

jcranmer 5y ago

> Better would be for the only role of the FDA in decisions like this to be determining if the manufacturer is making fraudulent claims. Let us recall some of the key facts about the drug in question here. There were two trials. One of them showed such little benefit that it was halted midtrial on the basis that it would be unethical to continue giving people a drug that didn't work. The second showed marginal benefit. Biogen argued that there is a rational explanation that explains the two trials that points to the drug working. The expert committee at the FDA had 10 people vote "that's bunk", 1 person vote "unsure", and nobody vote "I agree." That's a pretty strong endorsement for saying "Aduhelm treats Alzheimer's" is a fraudulent claim, and yet the FDA decided that the claim is merely disputed. The only way you can look on the FDA's decision favorably is if you know nothing about how drug development works, which is unfortunately true of almost the entire public. (Here's two facts that are important to keep in mind: most drug candidates don't work, and placebo effects are very real and surprisingly powerful).

mcguire 5y ago

I suppose there is some fine ethical reasoning that would distinguish between selling something that you know not to work and selling something that you don't know won't work. Of course, financially incentivizing ignorance is probably not the best precedent.