6 ms·

The one silver lining of having such a terrible diagnosis should be that you are immediately unbound from normal FDA requirements and are able and try anything

The one silver lining of having such a terrible diagnosis should be that you are immediately unbound from normal FDA requirements and are able and try anything in the pipeline that might work.

rfrey 2y ago
With the caveat that they can only be charged a nominal fee, say the actual production cost of the drug. It would be good to avoid fraudsters stripping desperate people if all their assets
- danielmarkbruce 2y ago
  This is an hilariously straightforward and brilliant idea. It's no holds barred, but the goal has got to be a real, approved drug in the future. Not profits right now.
- cogman10 2y ago
  I'd probably go further and say it must be free of charge. The problem with a nominal fee is fraudsters can be really clever at finding ways to bump up production costs. You might give the company doing the research relaxed liability, but even then you'd want to be careful. After all, you wouldn't want a coal mining operation conducting "research on lung cancer treatment" by sending in miners without PPE. If this is a legitimate research into curing cancer then eating the cost to treat test subjects is the least a company could do. The results are what's can be worth a lot of money.
  - cogman10 2y ago
    Actually, thinking about this further, even free of charge could be problematic. I can 100% see a "cancer research and spiritual healing" clinic popping up that uses the promise is a cure as a way to get someone in the door so they can later be sold on energy healing to increase effectivity of the sugar pill they give away. Fraudsters simply love preying on the desprate.
    - ToValueFunfetti 2y ago
      Would it be illegal today to give people sugar pills sell them energy healing?
      - cogman10 2y ago
        Ish. You can't say that a pill or treatment cures cancer if there's no evidence to back that up. Of course in private I'm sure that's said all the time. My concern with lowering the bar for advertising such services is it makes it easier for someone to jump in with "Our clinic is actively researching cancer treatments and offers free experimental drugs".
      - IX-103 2y ago
        Depends on how it's marketed. As long as they avoid making specific health claims (say "boost immune function" instead of "cures disease X") they can get away with it. If it's "homeopathic" they can even be sold on the same shelves as actual medicine, with similar looking labels.
  - TZubiri 2y ago
    I don't see the issue or point of limiting profitabilit, what am I missing? Here's the scenario I have in mind. John has an uncommon cancer X with no cure. John contacts Cancer R&D Corp. John pays 80% of his life savings to R&D corp for research into his specific cancer X and provides himself as a test subject in the meantime. R&D corp attempts to find a cure for the disease, if the mission fails and John dies, in the future if other patients with cancer X appear, they can take over from where they left. Payment can be split into an upfront half for research and bonus paid upon a successful cure (or year of quality life milestones) to give incentives for actual success. So as to avoid companies from benefitting from never finding a cure.
    - fwip 2y ago
      To see the issue, replace "R&D Corp" with "Mystical Crystal LLC" or "Mr. Henry's Miraculous Snake Oil Elixir Ltd." Fraudsters and scam artists are happy to rip off people trying to avoid death.
      - JumpCrisscross 2y ago
        Or Steve Jobs. The problem isn't so much that the treatments don't work. It's that they're pursued exclusive of ones that do.
      - TZubiri 2y ago
        I responded to this in other posts, but this would be throwing the baby with the bath water. And it's especially bad as the good suffers more. Legitimate companies will follow the law, but the ilegitimate actors will just sell mystical crystal anyways.
        fwip 2y ago
        I've never really understand this whole "laws are useless because criminals will break them" thing.
    - cogman10 2y ago
      The issue is that fraudsters would like nothing more than to call themselves R&D and to sell BS to desperate people looking for a cure. That already happens very frequently. For example, bleach enemas to cure autism [1]. Colloidal metals to cure cancer [2]. And a whole host of other cure-alls. Now imagine you have a mechanism where they can claim to be legitimate research AND there is an incentive to bilk people out of 80% of their life savings. Heck, even imagine what happens if legitimate for profit companies can use this route for revenue generation. If they have a route to sell drugs which has less regulation and liability, why would they ever release a drug through the full FDA process? And why, you might ask, is regulation needed for this sort of stuff? Well, think Vioxx.[3] Sure, maybe this cancer drug works but it also might give you a heart attack. Without getting it fully approved or researched drug manufacturers have no reason to actually look into downstream effects. They have every incentive to just keep it on the market (like they did with Vioxx) and ignore evidence of adverse results. Taking profit out of the equation makes it so that the research isn't and can't be endless. It makes sure there isn't a perverse incentive to make lethal drugs that work good enough for some diseases. I'd love it if we could have a win win, but the free market loves to reward bad actors. [1] https://www.nbcnews.com/tech/internet/moms-go-undercover-fight-fake-autism-cures-private-facebook-groups-n1007871 https://www.nbcnews.com/tech/internet/moms-go-undercover-fig... [2] https://www.healthline.com/health/colloidal-silver-cancer https://www.healthline.com/health/colloidal-silver-cancer [3] https://en.wikipedia.org/wiki/Rofecoxib https://en.wikipedia.org/wiki/Rofecoxib
  - ChadNauseam 2y ago
    I could be wrong, but my understanding is that "free of charge" is already legal, and the FDA only regulates drugs that are sold and not ones that are given freely
    - cogman10 2y ago
      I'm not sure how the law pays with free goods. I know that a drug company can't skirt regulations around clinical trials simply by making participation free (in fact, clinical trials usually pay the subjects.)
- n4r9 2y ago
  I think you also need a caveat that failures must be recorded, and a specific treatment can only be used so many times without success. That will avoid providrrs who are self-deluded about the efficacy of their favourite drug/therapy.
- TZubiri 2y ago
  Conversely, if you ban actually charging for last-attempt experimental research services, then those desperate people will only be left to seek: 1- Honest researchers who can't charge too much. 2- Unscrupulous quacks who will take all your money regardless of law. With the second option being much more common due to incentives. If however you allow research facilities to take donations/payments to fund research related to a specific disease, then you will increase the incentive for legitimate companies, even if the odds are still low or close to 0.
y-c-o-m-b 2y ago
We are getting somewhat closer with "Right-to-try" laws: https://en.wikipedia.org/wiki/Right-to-try_law https://en.wikipedia.org/wiki/Right-to-try_law Jake Seliger (RIP) wrote about his frustrations with it also: https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-cell-carcinoma-and-the-treatments-that-might-save-me-are-just-out-of-reach/ https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-ce... https://jakeseliger.com/2023/08/02/if-youre-involved-in-drug-development-and-have-first-hand-knowledge-of-the-fdas-torpor-get-in-touch/ https://jakeseliger.com/2023/08/02/if-youre-involved-in-drug...
- directevolve 2y ago
  https://en.wikipedia.org/wiki/Right_to_repair https://en.wikipedia.org/wiki/Right_to_repair
- bsder 2y ago
  Jake Seliger's problems weren't really "Right-to-try". The first problem was finding the damn trial in the first place since nobody wants to share information as it might tell their competitors something. And then he had to pass the prerequisites for the trial. The problem there was navigating the US health system that moves like a glacier and either couldn't get a test run fast enough or wouldn't take a test from somewhere else. Finally, after all that, then you reached the "Right-to-try" level where there were different questions of whether to subject him to the treatment based upon whether the doctor thought it might or might not be effective. And through it all, he and his wife understood that the odds were very, very bad. To me, the counterargument to "Right-to-try" are the Alzheimer's drugs. The underlying Amyloid hypothesis appears to be broken and fraudulent and all the drugs based on it have failed miserably. And, yet, one got through FDA approval because the consumer push is so huge and everybody wants something--even if it doesn't work. There is big money behind pushing the boundaries--be very careful about eroding them.
  - spott 2y ago
    To me, the Alzheimer’s drug story is an argument for right to try laws. If the drug is available to the desperate, then the push by the public to shortcut the process goes away and the process can be done correctly. Those who are desperate can try, with the understanding that it might not work. The process can continue unmodified because there isn’t a pressure to get the drug out to the public because it is available.
pfisherman 2y ago
The “might work” is doing a lot of heavy lifting in this sentence. How strong must the evidence be for the hypothesis of efficacy to be considered reasonable?
- anamexis 2y ago
  Why not leave that question to the person getting treated?
  - lucianbr 2y ago
    The person being treated, or their family, might change their mind after the treatment fails, and cry bloody murder. And get sympathy. So the only defense is to deny treatment. In hindsight, with the bad consequences fixed, they will claim they should have been prevented from getting the treatment and whoever did not prevent them is at fault. Do you doubt there are people who refuse to accept the consequences of their own actions and seek to place blame anywhere else they can? Or indeed some third party might do the claim with hindsight. Prosecutors or whatever. People can be irrational, in general.
    - anamexis 2y ago
      Those all sound like legislative issues that could be fixed by the legislature enabling these kinds of experimental treatments in the first place.
      - throwaway2037 2y ago
        I find this comment somewhat disingenuous. People have been arguing about allowing more experimental end of life treatments for at least 40 years of my life. I grew up during the AIDS crisis and there were all kinds of quacks selling this or that to take easy money from desperate people. If what you say is true, then why hasn't it already happened? After all, isn't 40+ years long enough to argue about it?
        2y ago
syndicatedjelly 2y ago
Are companies similarly unbound from advertising anything they want to vulnerable populations? What do those rules look like?
redleader55 2y ago
Patients with incurable or hard to cure diseases are susceptibile to be exploited by companies or doctors that want to try unproven treatments on them. On the other hand, telling someone they are not allowed to do everything they can to save themselves is equally bad.
- RockRobotRock 2y ago
  How can we collect useful data on experimental medicine outside of a controlled study?
  - dredmorbius 2y ago
    Centres of disease control (in the US and elsewhere) track morbidity and mortality on a wide range of conditions, which is one standardised answer to your question. There are also (a small number of) independent or unorthodox researchers who pursue specific avenues. You'll of course find the frauds amongst these (e.g., Andrew Wakefield, who promulgated anti-vax theories and falsified research toward same), but there are also notable medical advances which have emerged in such contexts, such as J. Robin Warren and Barry Marshall's identification of Helicobacter pylori as the principle cause of stomach ulcers, rather than stress: Jessica Tanenbaum, "Delayed Gratification: Why it Took Everybody So Long to Acknowledge that Bacteria Cause Ulcers" (2005) <https://www.jyi.org/2005-february/2005/2/9/delayed-gratification-why-it-took-everybody-so-long-to-acknowledge-that-bacteria-cause-ulcers https://www.jyi.org/2005-february/2005/2/9/delayed-gratifica...>
bsder 2y ago
"Bleach cures Covid". Now what? The irrational FAR outnumber the irrational.
- vdvsvwvwvwvwv 2y ago
  It sounds like this might be a bit like off-label medicine though. It would need to start with a doctor. Maybe any plan needs an independent sign off from another doctor who is qualified enough.
deleted 2y ago
[deleted]
silexia 2y ago
How about if the government doesn't tell us what to do with our bodies to start with?

The one silver lining of having such a terrible diagnosis should be that you are immediately unbound from normal FDA requirements and are able and try anything in the pipeline that might work.

rfrey 2y ago

With the caveat that they can only be charged a nominal fee, say the actual production cost of the drug. It would be good to avoid fraudsters stripping desperate people if all their assets

danielmarkbruce 2y ago
This is an hilariously straightforward and brilliant idea. It's no holds barred, but the goal has got to be a real, approved drug in the future. Not profits right now.
cogman10 2y ago
I'd probably go further and say it must be free of charge. The problem with a nominal fee is fraudsters can be really clever at finding ways to bump up production costs. You might give the company doing the research relaxed liability, but even then you'd want to be careful. After all, you wouldn't want a coal mining operation conducting "research on lung cancer treatment" by sending in miners without PPE. If this is a legitimate research into curing cancer then eating the cost to treat test subjects is the least a company could do. The results are what's can be worth a lot of money.
- cogman10 2y ago
  Actually, thinking about this further, even free of charge could be problematic. I can 100% see a "cancer research and spiritual healing" clinic popping up that uses the promise is a cure as a way to get someone in the door so they can later be sold on energy healing to increase effectivity of the sugar pill they give away. Fraudsters simply love preying on the desprate.
  - ToValueFunfetti 2y ago
    Would it be illegal today to give people sugar pills sell them energy healing?
    - cogman10 2y ago
      Ish. You can't say that a pill or treatment cures cancer if there's no evidence to back that up. Of course in private I'm sure that's said all the time. My concern with lowering the bar for advertising such services is it makes it easier for someone to jump in with "Our clinic is actively researching cancer treatments and offers free experimental drugs".
    - IX-103 2y ago
      Depends on how it's marketed. As long as they avoid making specific health claims (say "boost immune function" instead of "cures disease X") they can get away with it. If it's "homeopathic" they can even be sold on the same shelves as actual medicine, with similar looking labels.
- TZubiri 2y ago
  I don't see the issue or point of limiting profitabilit, what am I missing? Here's the scenario I have in mind. John has an uncommon cancer X with no cure. John contacts Cancer R&D Corp. John pays 80% of his life savings to R&D corp for research into his specific cancer X and provides himself as a test subject in the meantime. R&D corp attempts to find a cure for the disease, if the mission fails and John dies, in the future if other patients with cancer X appear, they can take over from where they left. Payment can be split into an upfront half for research and bonus paid upon a successful cure (or year of quality life milestones) to give incentives for actual success. So as to avoid companies from benefitting from never finding a cure.
  - fwip 2y ago
    To see the issue, replace "R&D Corp" with "Mystical Crystal LLC" or "Mr. Henry's Miraculous Snake Oil Elixir Ltd." Fraudsters and scam artists are happy to rip off people trying to avoid death.
    - JumpCrisscross 2y ago
      Or Steve Jobs. The problem isn't so much that the treatments don't work. It's that they're pursued exclusive of ones that do.
    - TZubiri 2y ago
      I responded to this in other posts, but this would be throwing the baby with the bath water. And it's especially bad as the good suffers more. Legitimate companies will follow the law, but the ilegitimate actors will just sell mystical crystal anyways.
      - fwip 2y ago
        I've never really understand this whole "laws are useless because criminals will break them" thing.
  - cogman10 2y ago
    The issue is that fraudsters would like nothing more than to call themselves R&D and to sell BS to desperate people looking for a cure. That already happens very frequently. For example, bleach enemas to cure autism [1]. Colloidal metals to cure cancer [2]. And a whole host of other cure-alls. Now imagine you have a mechanism where they can claim to be legitimate research AND there is an incentive to bilk people out of 80% of their life savings. Heck, even imagine what happens if legitimate for profit companies can use this route for revenue generation. If they have a route to sell drugs which has less regulation and liability, why would they ever release a drug through the full FDA process? And why, you might ask, is regulation needed for this sort of stuff? Well, think Vioxx.[3] Sure, maybe this cancer drug works but it also might give you a heart attack. Without getting it fully approved or researched drug manufacturers have no reason to actually look into downstream effects. They have every incentive to just keep it on the market (like they did with Vioxx) and ignore evidence of adverse results. Taking profit out of the equation makes it so that the research isn't and can't be endless. It makes sure there isn't a perverse incentive to make lethal drugs that work good enough for some diseases. I'd love it if we could have a win win, but the free market loves to reward bad actors. [1] https://www.nbcnews.com/tech/internet/moms-go-undercover-fight-fake-autism-cures-private-facebook-groups-n1007871 https://www.nbcnews.com/tech/internet/moms-go-undercover-fig... [2] https://www.healthline.com/health/colloidal-silver-cancer https://www.healthline.com/health/colloidal-silver-cancer [3] https://en.wikipedia.org/wiki/Rofecoxib https://en.wikipedia.org/wiki/Rofecoxib
- ChadNauseam 2y ago
  I could be wrong, but my understanding is that "free of charge" is already legal, and the FDA only regulates drugs that are sold and not ones that are given freely
  - cogman10 2y ago
    I'm not sure how the law pays with free goods. I know that a drug company can't skirt regulations around clinical trials simply by making participation free (in fact, clinical trials usually pay the subjects.)
n4r9 2y ago
I think you also need a caveat that failures must be recorded, and a specific treatment can only be used so many times without success. That will avoid providrrs who are self-deluded about the efficacy of their favourite drug/therapy.
TZubiri 2y ago
Conversely, if you ban actually charging for last-attempt experimental research services, then those desperate people will only be left to seek: 1- Honest researchers who can't charge too much. 2- Unscrupulous quacks who will take all your money regardless of law. With the second option being much more common due to incentives. If however you allow research facilities to take donations/payments to fund research related to a specific disease, then you will increase the incentive for legitimate companies, even if the odds are still low or close to 0.

y-c-o-m-b 2y ago

We are getting somewhat closer with "Right-to-try" laws: https://en.wikipedia.org/wiki/Right-to-try_law https://en.wikipedia.org/wiki/Right-to-try_law Jake Seliger (RIP) wrote about his frustrations with it also: https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-cell-carcinoma-and-the-treatments-that-might-save-me-are-just-out-of-reach/ https://jakeseliger.com/2023/07/22/i-am-dying-of-squamous-ce... https://jakeseliger.com/2023/08/02/if-youre-involved-in-drug-development-and-have-first-hand-knowledge-of-the-fdas-torpor-get-in-touch/ https://jakeseliger.com/2023/08/02/if-youre-involved-in-drug...

directevolve 2y ago
https://en.wikipedia.org/wiki/Right_to_repair https://en.wikipedia.org/wiki/Right_to_repair
bsder 2y ago
Jake Seliger's problems weren't really "Right-to-try". The first problem was finding the damn trial in the first place since nobody wants to share information as it might tell their competitors something. And then he had to pass the prerequisites for the trial. The problem there was navigating the US health system that moves like a glacier and either couldn't get a test run fast enough or wouldn't take a test from somewhere else. Finally, after all that, then you reached the "Right-to-try" level where there were different questions of whether to subject him to the treatment based upon whether the doctor thought it might or might not be effective. And through it all, he and his wife understood that the odds were very, very bad. To me, the counterargument to "Right-to-try" are the Alzheimer's drugs. The underlying Amyloid hypothesis appears to be broken and fraudulent and all the drugs based on it have failed miserably. And, yet, one got through FDA approval because the consumer push is so huge and everybody wants something--even if it doesn't work. There is big money behind pushing the boundaries--be very careful about eroding them.
- spott 2y ago
  To me, the Alzheimer’s drug story is an argument for right to try laws. If the drug is available to the desperate, then the push by the public to shortcut the process goes away and the process can be done correctly. Those who are desperate can try, with the understanding that it might not work. The process can continue unmodified because there isn’t a pressure to get the drug out to the public because it is available.

pfisherman 2y ago

The “might work” is doing a lot of heavy lifting in this sentence. How strong must the evidence be for the hypothesis of efficacy to be considered reasonable?

anamexis 2y ago
Why not leave that question to the person getting treated?
- lucianbr 2y ago
  The person being treated, or their family, might change their mind after the treatment fails, and cry bloody murder. And get sympathy. So the only defense is to deny treatment. In hindsight, with the bad consequences fixed, they will claim they should have been prevented from getting the treatment and whoever did not prevent them is at fault. Do you doubt there are people who refuse to accept the consequences of their own actions and seek to place blame anywhere else they can? Or indeed some third party might do the claim with hindsight. Prosecutors or whatever. People can be irrational, in general.
  - anamexis 2y ago
    Those all sound like legislative issues that could be fixed by the legislature enabling these kinds of experimental treatments in the first place.
    - throwaway2037 2y ago
      I find this comment somewhat disingenuous. People have been arguing about allowing more experimental end of life treatments for at least 40 years of my life. I grew up during the AIDS crisis and there were all kinds of quacks selling this or that to take easy money from desperate people. If what you say is true, then why hasn't it already happened? After all, isn't 40+ years long enough to argue about it?
      - 2y ago

syndicatedjelly 2y ago

Are companies similarly unbound from advertising anything they want to vulnerable populations? What do those rules look like?

redleader55 2y ago

Patients with incurable or hard to cure diseases are susceptibile to be exploited by companies or doctors that want to try unproven treatments on them. On the other hand, telling someone they are not allowed to do everything they can to save themselves is equally bad.

RockRobotRock 2y ago
How can we collect useful data on experimental medicine outside of a controlled study?
- dredmorbius 2y ago
  Centres of disease control (in the US and elsewhere) track morbidity and mortality on a wide range of conditions, which is one standardised answer to your question. There are also (a small number of) independent or unorthodox researchers who pursue specific avenues. You'll of course find the frauds amongst these (e.g., Andrew Wakefield, who promulgated anti-vax theories and falsified research toward same), but there are also notable medical advances which have emerged in such contexts, such as J. Robin Warren and Barry Marshall's identification of Helicobacter pylori as the principle cause of stomach ulcers, rather than stress: Jessica Tanenbaum, "Delayed Gratification: Why it Took Everybody So Long to Acknowledge that Bacteria Cause Ulcers" (2005) <https://www.jyi.org/2005-february/2005/2/9/delayed-gratification-why-it-took-everybody-so-long-to-acknowledge-that-bacteria-cause-ulcers https://www.jyi.org/2005-february/2005/2/9/delayed-gratifica...>

bsder 2y ago

"Bleach cures Covid". Now what? The irrational FAR outnumber the irrational.

vdvsvwvwvwvwv 2y ago
It sounds like this might be a bit like off-label medicine though. It would need to start with a doctor. Maybe any plan needs an independent sign off from another doctor who is qualified enough.

deleted 2y ago

[deleted]

silexia 2y ago

How about if the government doesn't tell us what to do with our bodies to start with?