Comment by seanmcdirmid
3 years ago
They had this debate in China recently and the problem was conflict of interest (lots of pricey snake oil for dying patients who can come up with the money). The market has to be regulated even for dying people, if only to prevent scams.
Perhaps the patients should be allowed to use experimental treatments only under the condition that it is offered for free; the upside for the company is that if it works, they get the evidence of efficacy.
That would be the bare minimum in practice. That’s already the case: without premarketing approval (clinical trials), companies are not allowed to charge anything for medicine or medical devices. They’re not even legally allowed to ship them interstate until the premarketing application is accepted.
This should be the solution. If they’re trials for an unproven solution, why should they pay anything at all. I’m any case, it should be the other way around; it’s the companies who should pay the person for subjecting them to something very likely to be dangerous and painful.
In any case, I agree with the sentiment here. People should have the right to choose.
This is how it already works - this is what a clinical trial is. If you want to include the data point of a medicine working well as admissible, then you better have more than one patient try it at a time. Unless of course your drug is so good at curing patients who are absolutely terminal (like ibrutinib was). But most meds are not that much of a blockbuster. Thus you don’t know if a single patient survived if it’s because this medicine was very effective or if it was just as good as any other available treatment regimen.
>This is how it already works - this is what a clinical trial is.
That's not at all how clinical trials work. If and when someone decides to advance a drug to the next stage of development, it's done because they think there's a sufficiently profitable market at the end of the road. So lots of extremely promising drugs stall out if the numbers don't make sense. Furthermore, the trial enrollment process itself is cumbersome and often your only entrypoint is email "trials@pharmacompany.com" and hope you can a response and can navigate their arbitrary screening process. Speaking of screening: trials are full of exclusion criteria. They can get copy-pasted between trial protocols and often have no real reason excluding you. What's the washout period for your last line of chemo? Ever had any kind of immunotherapy? &c &c From the point of view of whoever is trying to get a drug approved it's better to be safe than sorry wrt any kind of uncertainty in a patient's medical status or treatment history. But that means that there might be a drug that looks extremely promising from previous trials but the only current trial excludes you for a small reason.
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The problem with clinical trials is that they are looking for specific patients, though. And they will exclude patients that introduce confounding variables. Some studies exclude patients that are closest to death because they will interfere with the trial as well.
> the upside for the company is that if it works, they get the evidence of efficacy.
They don't, though. They need a control group.
> They don't [get evidence of efficacy], though. They need a control group.
This is false. Control groups, preferably in double-blind studies, are an essential part of best practices. However, the bar for what constitutes evidence is much lower than that.
Evidence is any fact that makes a relevant conclusion more or less likely. Evidence need not be conclusive in and of itself. Over time, the cumulative effects of less-strong evidence can form the basis for a reasonable inductive conclusion. Sorry to be trite, but as an example, a person can have evidence that their romantic partner loves them without the use of an identical twin.
Insisting on gold standard medical evidence before allowing someone to undergo life-saving treatment—when the preponderance of the evidence that does exist supports that treatment—is, to be blunt, the very definition of depraved heart murder. It demonstrates a wanton disregard toward human life in the face of the known likelihood that it will result in some number of deaths.
More broadly, however, it is an extremely poor policy, in that this meme both weakens society's critical thinking generally and—among those who see through the elitist charade—significantly weakens the respect of those scientists who make the claim.
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The people who have already died to-date of your terminal illness aren't a good enough control group to tell that your cure works? This is the absurdity of frequentist statistics. If you can't tell the difference between recovering from the treatment and recovering by natural luck, then either the treatment is barely effective at all, or the doctor had no basis to tell you in the first place that you had a terminal illness.
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There is a standard of evidence above guesswork and below controlled trials, that is still useful: you accumulate enough survival rate data of whatever specific terminal condition you're hoping to treat, and compare it to the baseline untreated survival rate, which is already known.
Any statistically significant difference there can then incentivize a drug research company to invest in the (expensive) clinical controlled trial process.
It's strange that people here are actually saying it's better to waste perfectly good research opportunities and watch patients die, than to risk having anything less than perfect evidence of efficacy.
And that is why you shouldn't be able to ask for the experimental treatment... You should be able to ask to be part of a trial of the experimental treatment.
Remember that if 10 treatments are being tried at once, you can divide the pool of participants so that only a smallish percentage are part of the control group.
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They don’t exactly need a control group if the chance of living N weeks is effectively zero. They would need it for something like living N weeks and living N+1 weeks, but any kind of change from an individual expected outcome would be useful signal for maybe more rigorous testing.
Not always. In some cases, like rabies, the control group is 100% rate of death.
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I think it’s common practice to give life saving treatment to the control group after there is some measure of confidence that the drug works.
They can then apply to launch formal clinical trials with a control group. This initial evidence of efficacy is of-course anecdotal evidence with more traceability.
Meh, why not just let some people get fleeced...?
"Sorry, you can't buy cars privately any more -- you need a gov't agency to make sure you don't get screwed. You cannot fend for yourself, right?"
Would you feel the same if they got an "experimental treatment" from some quack who ended up giving them a highly dangerous chemical which resulted in a slow and extremely painful death? Because that is 100% what is going to happen when you remove all regulations for experimental medicine.
Similarly, you already cannot buy all cars privately anymore. Business-to-consumer is highly regulated (that's what the Federal Motor Vehicle Safety Standards are for, but home-built cars also need to pass several inspections before they are allowed on the road.
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I know the reason, and it’s a doozy. Send me $5 and I’ll let you in on the secret.
This sounds like the best way to do it.
The company would still need a blinded control group to be able to demonstrate the effectiveness of any treatment.
However patients should be allowed to enroll for free into multiple treatment trials at the same time. For a given number of people and treatments, enrolling in multiple trials simultaneously gives more science output, and more chance of success for the participants, if you assume that ineffective treatments rarely are very harmful.
"However patients should be allowed to enroll for free into multiple treatment trials at the same time."
But this would likely mess up any data, you can get. How can you tie the cure or the bad sideeffects to any drug then?
Apart from that, sure, I think patients should have the right to try anything.
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I was going to make this point. People at EOL by default often have estates, investments, retirement accounts. In other words huge chunks of money/wealth that unscrupulous people may wish to get their hands on. Combine that with the desperation of a dying person and you have the perfect condition to create a very profitable scammer market for worthless treatments.
This also harms eveyone because all the money pouring into a treatment that was a scam from day one is oppertunitu y lost.
> This also harms eveyone because all the money pouring into a treatment that was a scam from day one is oppertunitu y lost.
You guys remember Theranos? When Safeway announced their deal with Theranos in 2012, a bunch of companies working in blood diagnostics folded because they couldn’t raise money or because their investors clawed back entire rounds.
A couple of friends had just raised a series B round to commercialize their surface plasmon resonance blood testing device, which would have actually done what Theranos promised with onsite blood testing (except with proper blood draw, not a finger prick). They were getting the product ready for golden standard testing with a CRO when investors decided it wasn’t worth trying to compete with a Theranos already in the pocket of major pharmacies and pulled the series B funding.
Patents ended up getting sold to some patent troll shortly before the news broke that Theranos was a scam.
The people who funded Theranos weren’t even desperate, just greedy. Imagine what fingers they’ll put on the scales when they’re actually staring death right in the face? It’ll be a disaster.
I think the argument is to loosen the regulation on human trials, not to drop all regulation on drug companies. They should still have to prove that they’re doing legitimate research and have had some success before opening it up to humans who are terminally ill. They should still be legally culpable for negligence and fraud.
Keep in mind that it's not the pharma companies that are the only, or even most notorious, scammers that would benefit. Families of the dying one are a major risk factor. The difficult[0] truth is, plenty of families would accelerate the demise of a terminally ill or elderly[1] if it were possible. Like with euthanasia, any elective experimental treatment that carries increased risk of death, could become something the patients get pressured or manipulated into by their families.
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[0] - The situations can be ethically and emotionally complex, so I'm not passing any generalized judgement, other that this is something a person might be concerned about when contemplating the possibility of ending up in this situation in the future.
[1] - Arguably the same thing, but that's another topic.
As a general principle, any set of circumstances which results in net increased influence of people who are demonstrably amoral with regard to the public welfare, should have as many barriers as possible put up to obstruct the success of such people.
Bad money chases out good, increasing the fraction of money in bad hands by billions or tens of billions is going to have a palpable effect on the stability of the world.
This is a very weak excuse. If preventing scams is the main concern then old people should also be banned from purchasing real estate, timeshares, large donations, perhaps even giftcards in certain quantities.
You can't have freedom without some chance of people making bad choices.
Dad asks me for a lot of money to help him buy a time share, I’ll feel ok saying no.
Dad asks me for a lot of money for a snake oil cure that might prevent him from dying from bile duct cancer in a month or two…how am I going to say no to that?
"No dad, it's a scam; there's no evidence, 100 people have taken it with no success, etc."
I have a severely disabled dad and have had this convo.
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Which is why there needs to be a process to approve experimental drugs as at least scientifically plausible.
There's a difference between something that works at least in a petri dish or mice, and something with no plausible mechanism of action like homoeopathy.
I guess it would depend on the reason for denial to a patient. If it is solely because it isn't regulatory approved yet then that doesn't seem sufficient. If the drug was provided for free from the pharma company it would be a faster path to a human trial which would be a win/win for all involved with the only possible negative for a person who doesn't have anything to lose.
That seems like a claims and marketing issue, which is entirely separate from merely producing a drug and selling it when asked. I think it's fine that the FDA regulates advertising, I don't think it's fine that they act as gatekeepers to pharmaceutical products.
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Ahhh. This answers a question I've had: I'm in the USA and one of my senators has been banging the drum for allowing patients access to experimental drugs for a few years. I have wondered why this was an issue for him. Maybe I'm cynical but this and easy PR win for him seems like good reasons why.
If they can come up with the money then who cares?
It would be a much better case for regulating if there were some external payer.
Their parents and/or kids come up with money. It is basically theft. If you are ok with theft, fine, otherwise we shouldn’t exempt it just because of the false hope it provides.
Did the Chinese government ultimately regulate the market to protect people and prevent this kind of corruption?
This wiki article is probably the best place to start:
https://en.m.wikipedia.org/wiki/Death_of_Wei_Zexi
Then there was some movement on the issue in 2019:
https://www.bloomberg.com/news/features/2019-12-10/cancer-tr...
I’m not sure where they ended up due to COVID happening next.
IIRC it did, though the cynic in me would suggest that by “this kind of corruption” what it prevents is the kind of corruption that government officials don't benefit from.
I trust the Chinese government less than I trust my own with regard to doing the right thing (just because it is the right thing or indeed at all), and that is a pretty low bar…
The regulation is approval by a governmental agency through clinical trials
There are tradeoffs here, but the current approach seems to protect people's inheritances better than people's lives.
Selling straight up snake oil is still fraud, and letting money be thrown at overhyped but ineffective treatments is a smaller tragedy than forcing people to die when treatments exist.
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