6 ms·

I totally believe right to try should be a thing. Who is the FDA or anyone else to tell dying patients that they can't put something in their body to try to sav

I totally believe right to try should be a thing. Who is the FDA or anyone else to tell dying patients that they can't put something in their body to try to save their life? The reason this is necessary is because under the previous compassionate use policy, pharma conpanies are required to report any adverse events from these patients using these products to FDA which could result in a halt of their independent clinical trials running in parallel. Because the patients utilizing compassionate use many be in any stage of disease with multiple comorbidities, it makes root cause analysis of adverse events much more difficult than in a clinical trial, and presents a much higher risk to developers. This strongly disincentivizes companies from leveraging the compassionate use pipeline, and patients are left to die and suffer as a result. Also as far as profiteering goes, there is no way that an insurance company would reimburse for any untested therapy which by default would keep costs down, and patients would still be required to get a prescription from their physician who would hopefully review the trial data. The idea that this would be used as an end run around FDA regulation for huge profits is moronic.

kazinator 8y ago
People shouldn't be charged to serve as guinea pigs. On the contrary, they should be paid. Here is a problem: if a pharma company can simply make money from someone's desperation, they have a reduced incentive to actually make the drug work. The situation is reduced to a simple scam: the business model of selling some desperate people the promise of a cure which is just snake oil. We always have to consider the effect of how the flow of money in a situation creates incentives that have unintended consequences.
- briandear 8y ago
  But that should be their choice to make.
  - kazinator 8y ago
    Is that so? In that case, why should only the special class of the terminally ill have the privilege of this choice? Everyone should have it. Just do away with drug trials and approvals in favor of a situation whereby anyone can advertise any substance whatsoever as being a cure for any ailment. The free market will determine what drugs work, no problem! If that's not okay, why is okay in the case of a special class of patients? Because they will be dead soon anyway, so who cares?
  - rhizome 8y ago
    It's not an informed choice if the offerer is lying.
    - kazinator 8y ago
      The players who are initially in that game might not be lying; they may offer full disclosure about the treatments so everyone is informed about what their chances are and the risks and so on. But a "can of worms" is open: all sorts of scum will notice the money-making opportunity and want a piece of the action. The drug approval and regulation mechanism is what limits that sort of abuse. Remove it out of the way and the abuse will creep in.
      - rhizome 8y ago
        You can't lie about FDA approval without easily being found out. The drug approval and regulation mechanism is what limits that sort of abuse. Remove it out of the way and the abuse will creep in. Uhhh, I think you skipped over at least eight steps in your logic there.
- Thriptic 8y ago
  What are you talking about? Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is, not individual patients who can't pay. That requires FDA approval. Historically iirc compassionate use distributions were free, and it is likely that will continue to be the case because the amount of money companies can make off of individual patients is rounding error relative to bringing a product to market and getting widespread approval for reimbursement. Also, the whole point is that these people are not guinea pigs. They are allowed access to promising therapies in late stage clinical trials in the hopes that they will help them, not just random investigational compounds. Let's remember that these requests would require physician approval. The result of blocking this is to say that dying patients should just die, tough shit, but hey at least they will die with more money right?
  - openasocket 8y ago
    > Also, the whole point is that these people are not guinea pigs. They are allowed access to promising therapies in late stage clinical trials in the hopes that they will help them, not just random investigational compounds I'm not positive, but I think this only requires drugs to have passed Phase I clinical trials. And that, according to the article, NurOwn's Phase II trials showed it was unable to slow the progression of ALS compared to a placebo.
  - frgtpsswrdlame 8y ago
    >Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is, not individual patients. I mean that's wrong because the article directly states that they're charging lots of money for this therapy so that they have an incentive. If 'the real money' was in insurance at scale, why does NurOwn require an additional monetary incentive beyond free test cases? Obviously they care about being able to bill individual patients. Especially in right-to-try cases because the type of person willing to pay to try an experimental therapy is often the type of person who would hand you everything they own. >The result of blocking this is to say that dying patients should just die, tough shit, but hey at least they will die with more money right? Most of these people are going to die anyway and many will now die experiencing side effects they had no way of knowing about. They'll also leave their families nothing. Marketizing healthcare has already raced us to an ugly bottom. This law creates a new and even lower bottom to how pharmaceutical companies are able to take advantage of some of the most desperate people our society has. That's not a good thing.
- beavisthegenius 8y ago
  As someone who successfully tested and completed stem cell therapy to prevent an amputation, I don't care who gets paid. Fuck you to saying I can't try something when it's my bone getting cut off.
Alex3917 8y ago
> Who is the FDA or anyone else to tell dying patients that they can't put something in their body to try to save their life? The FDA doesn't prevent you from taking whatever drugs you want, they (previously) just prevented companies from selling and marketing unapproved drugs as treatments. The law has a misleading name, patients were already allowed to take any of these drugs.
mylons 8y ago
I like the idealized version of putting whatever I want in my body before I die in an attempt to extend my life. There are definitely snake oil salesmen out there, and I do believe there is a place for the FDA to get in their way as much as possible.
- RobertRoberts 8y ago
  There seems to be an overlapping conflict area between the freedom to be ripped off and the freedom to control my own medical care. I'd rather have more control over my life than less, even if it means there's risk. At least it's my choice which risk, not someone else’s.
  - Nursie 8y ago
    The converse to this, though, is that there are people who do get duped, and they get ripped off for hundreds of thousands of dollars. Especially where, for instance, there are sick kids involved. You see campaigns in newspapers and on social media, for donations so that some poor, dying child with untreatable cancer can be flown to Texas or Mexico, and they just have to have $150K for the treatment and drugs they need. They aren't medically trained and they can't spot the scam - hell, they don't want to spot the scam, they want their child to live, and who can blame them? They've already taken out as much debt as they can afford. Their families and close friends have probably done the same, and they're campaigning for the general public to chip in too, for a treatment that's effectively a fraud. It's heartbreaking. This is where we need someone to step in and prevent the behaviour.
    - RobertRoberts 8y ago
      Education is a partial cure for this, as well as the law pursuing thieves. But there will always be scams no matter how many laws you pass or protections put on people.
      - Nursie 8y ago
        Yes there will always be scams. That doesn't mean we should leave the field wide open and encourage scams, or create a legal landscape in which it is difficult to detect and close down such things. Education is probably not any sort of cure for this - the scams are often run by educated, authoritative people who offer a fake lifeline to parents who are almost beyond hope and who are certainly not rationally evaluating the options. There is a role for authorities here.
Nursie 8y ago
I'm not sure I have as much faith in humanity as you do. We already have examples of long-running medical scams that take the dying for all they're worth, take the kind-hearted who donate to their causes for all they're worth, and often leave families in debt, all for a treatment that is little more than a tissue of lies. I agree wholeheartedly that if there is a genuine treatment in development, and if someone is on the path to imminent death, and there's the possibility it might cure them, or significantly enhance the length and quality of the life they have left, then denying them access to it is unforgivably cruel. But I'm not sure how you filter out the charlatans that have fraudulent products in perpetual 'trials', who want to take people for everything they have (and far more).
mannykannot 8y ago
The FDA already had a mechanism for this purpose, as did 38 states. After the passing of this bill, Senator Ron Johnson, one of the bill's sponsors, took upon himself the privilege of explaining how it should be implemented to FDA Commissioner Scott Gottlieb (a Trump appointee), apparently because Gottlieb expressed excessive concern for patients' well-being. This raises the possibility that the bill's authors were coy about their intentions, perhaps to avoid them being scrutinized prior to its passage, and I hope the FDA will interpret it as written, and consistently with its broader responsibilities. https://arstechnica.com/science/2018/06/new-drug-access-law-intended-to-weaken-fda-according-to-lawmaker-behind-it/ https://arstechnica.com/science/2018/06/new-drug-access-law-...
jonhendry18 8y ago
" no way that an insurance company would reimburse for any untested therapy which by default would keep costs down" Wrong. The patients just get second mortgages and/or crowdfund it. And people contribute because they don't know they're paying for the person to be subjected to useless quackery.

kazinator 8y ago

People shouldn't be charged to serve as guinea pigs. On the contrary, they should be paid. Here is a problem: if a pharma company can simply make money from someone's desperation, they have a reduced incentive to actually make the drug work. The situation is reduced to a simple scam: the business model of selling some desperate people the promise of a cure which is just snake oil. We always have to consider the effect of how the flow of money in a situation creates incentives that have unintended consequences.

briandear 8y ago
But that should be their choice to make.
- kazinator 8y ago
  Is that so? In that case, why should only the special class of the terminally ill have the privilege of this choice? Everyone should have it. Just do away with drug trials and approvals in favor of a situation whereby anyone can advertise any substance whatsoever as being a cure for any ailment. The free market will determine what drugs work, no problem! If that's not okay, why is okay in the case of a special class of patients? Because they will be dead soon anyway, so who cares?
- rhizome 8y ago
  It's not an informed choice if the offerer is lying.
  - kazinator 8y ago
    The players who are initially in that game might not be lying; they may offer full disclosure about the treatments so everyone is informed about what their chances are and the risks and so on. But a "can of worms" is open: all sorts of scum will notice the money-making opportunity and want a piece of the action. The drug approval and regulation mechanism is what limits that sort of abuse. Remove it out of the way and the abuse will creep in.
    - rhizome 8y ago
      You can't lie about FDA approval without easily being found out. The drug approval and regulation mechanism is what limits that sort of abuse. Remove it out of the way and the abuse will creep in. Uhhh, I think you skipped over at least eight steps in your logic there.
Thriptic 8y ago
What are you talking about? Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is, not individual patients who can't pay. That requires FDA approval. Historically iirc compassionate use distributions were free, and it is likely that will continue to be the case because the amount of money companies can make off of individual patients is rounding error relative to bringing a product to market and getting widespread approval for reimbursement. Also, the whole point is that these people are not guinea pigs. They are allowed access to promising therapies in late stage clinical trials in the hopes that they will help them, not just random investigational compounds. Let's remember that these requests would require physician approval. The result of blocking this is to say that dying patients should just die, tough shit, but hey at least they will die with more money right?
- openasocket 8y ago
  > Also, the whole point is that these people are not guinea pigs. They are allowed access to promising therapies in late stage clinical trials in the hopes that they will help them, not just random investigational compounds I'm not positive, but I think this only requires drugs to have passed Phase I clinical trials. And that, according to the article, NurOwn's Phase II trials showed it was unable to slow the progression of ALS compared to a placebo.
- frgtpsswrdlame 8y ago
  >Pharma conpanies want therapies to work because billing insurance companies at scale is where the money is, not individual patients. I mean that's wrong because the article directly states that they're charging lots of money for this therapy so that they have an incentive. If 'the real money' was in insurance at scale, why does NurOwn require an additional monetary incentive beyond free test cases? Obviously they care about being able to bill individual patients. Especially in right-to-try cases because the type of person willing to pay to try an experimental therapy is often the type of person who would hand you everything they own. >The result of blocking this is to say that dying patients should just die, tough shit, but hey at least they will die with more money right? Most of these people are going to die anyway and many will now die experiencing side effects they had no way of knowing about. They'll also leave their families nothing. Marketizing healthcare has already raced us to an ugly bottom. This law creates a new and even lower bottom to how pharmaceutical companies are able to take advantage of some of the most desperate people our society has. That's not a good thing.
beavisthegenius 8y ago
As someone who successfully tested and completed stem cell therapy to prevent an amputation, I don't care who gets paid. Fuck you to saying I can't try something when it's my bone getting cut off.

Alex3917 8y ago

> Who is the FDA or anyone else to tell dying patients that they can't put something in their body to try to save their life? The FDA doesn't prevent you from taking whatever drugs you want, they (previously) just prevented companies from selling and marketing unapproved drugs as treatments. The law has a misleading name, patients were already allowed to take any of these drugs.

mylons 8y ago

I like the idealized version of putting whatever I want in my body before I die in an attempt to extend my life. There are definitely snake oil salesmen out there, and I do believe there is a place for the FDA to get in their way as much as possible.

RobertRoberts 8y ago
There seems to be an overlapping conflict area between the freedom to be ripped off and the freedom to control my own medical care. I'd rather have more control over my life than less, even if it means there's risk. At least it's my choice which risk, not someone else’s.
- Nursie 8y ago
  The converse to this, though, is that there are people who do get duped, and they get ripped off for hundreds of thousands of dollars. Especially where, for instance, there are sick kids involved. You see campaigns in newspapers and on social media, for donations so that some poor, dying child with untreatable cancer can be flown to Texas or Mexico, and they just have to have $150K for the treatment and drugs they need. They aren't medically trained and they can't spot the scam - hell, they don't want to spot the scam, they want their child to live, and who can blame them? They've already taken out as much debt as they can afford. Their families and close friends have probably done the same, and they're campaigning for the general public to chip in too, for a treatment that's effectively a fraud. It's heartbreaking. This is where we need someone to step in and prevent the behaviour.
  - RobertRoberts 8y ago
    Education is a partial cure for this, as well as the law pursuing thieves. But there will always be scams no matter how many laws you pass or protections put on people.
    - Nursie 8y ago
      Yes there will always be scams. That doesn't mean we should leave the field wide open and encourage scams, or create a legal landscape in which it is difficult to detect and close down such things. Education is probably not any sort of cure for this - the scams are often run by educated, authoritative people who offer a fake lifeline to parents who are almost beyond hope and who are certainly not rationally evaluating the options. There is a role for authorities here.

Nursie 8y ago

I'm not sure I have as much faith in humanity as you do. We already have examples of long-running medical scams that take the dying for all they're worth, take the kind-hearted who donate to their causes for all they're worth, and often leave families in debt, all for a treatment that is little more than a tissue of lies. I agree wholeheartedly that if there is a genuine treatment in development, and if someone is on the path to imminent death, and there's the possibility it might cure them, or significantly enhance the length and quality of the life they have left, then denying them access to it is unforgivably cruel. But I'm not sure how you filter out the charlatans that have fraudulent products in perpetual 'trials', who want to take people for everything they have (and far more).

mannykannot 8y ago

The FDA already had a mechanism for this purpose, as did 38 states. After the passing of this bill, Senator Ron Johnson, one of the bill's sponsors, took upon himself the privilege of explaining how it should be implemented to FDA Commissioner Scott Gottlieb (a Trump appointee), apparently because Gottlieb expressed excessive concern for patients' well-being. This raises the possibility that the bill's authors were coy about their intentions, perhaps to avoid them being scrutinized prior to its passage, and I hope the FDA will interpret it as written, and consistently with its broader responsibilities. https://arstechnica.com/science/2018/06/new-drug-access-law-intended-to-weaken-fda-according-to-lawmaker-behind-it/ https://arstechnica.com/science/2018/06/new-drug-access-law-...

jonhendry18 8y ago

" no way that an insurance company would reimburse for any untested therapy which by default would keep costs down" Wrong. The patients just get second mortgages and/or crowdfund it. And people contribute because they don't know they're paying for the person to be subjected to useless quackery.