Comment by mitthrowaway2
3 years ago
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.
3 years ago
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.
I assume they meant that clinical trials are already free for the patient, as suggested by the comment they were responding to.
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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.
> Control groups, preferably in double-blind studies, are an essential part of best practices.
I wouldn't go that far. I think that a paradigm built upon a broader evidence base -- e.g. with far more extensive postmarketing surveillance of drugs -- would be more ethical, of more benefit to society, and ultimately more effective.
Double-blinded studies can miss, can be confounded by placebo effects, can easily be confounded by crooked researchers, and are frequently just plain unethical from first principles. I don't think that they should be considered "essential."
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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.
That is correct, the people that have already died are unable to act as effective controls. To establish the medicine causes the disease to go away, you have to use something like a randomized trial. Otherwise, causation is not established. It’s a very difficult problem and a lot of smart people have try to come up with better systems.
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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.
...I think that's basically how it works? If the company is running a trial, you can enroll in the trial. AFAIK being in a trial is free for participants.
Of course, there are any number of reasons someone may be rejected from the trial. If that happens, the company isn't going to just freely give them the drug anyway, because it wouldn't provide scientifically valid data. Also, no one would enroll in a trial (which carries the potential of getting the placebo) if they could get the treatment outside the trial.
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I have no better words: that is evil.
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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.
It is not. There are known treatment protocols with a reasonable (25%-ish) survival rate. There is also strong evidence that some people simply develop antibodies for it, and survive it without a vaccination or any treatment at all. It is rare, but definitely possible.
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.
No, not really. That's life... you're taking a risk.
Taking a risk just driving a car, I don't get why you can't take a risk with a medicine.
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.
You just end up with a matrix of treatments. Whatever results you observe from someone undergoing treatments X & Y goes in the "X&Y" box, not the X box and the Y box.
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Bad side effects caused by participating in other trials would happen with equal frequencies in the control group.