Comment by melling
3 years ago
This has been a common complaint for decades. We always want to try rush experimental drugs/procedures that “show promise”.
How often do these drugs, etc turn out to effective? 25 years ago we wanted to rush gene therapy and that didn’t turn out well. Someone died. It probably set back gene therapy:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC81135/
I think the solution is to invest more into medical research now, then when we, or someone we know, eventually ends up with some horrible disease, (cancer(s), Alzheimer’s, Parkinson’s, etc) we’ll know that we at least we made significant effort, and gave everyone a better chance, rather than settle for a “Hail Mary” drug near the end.
>I think the solution is to invest more into medical research now,
The author is arguing that terminally ill people (with a few months left to live) -- like him -- would be willing participants in trials to help medical research. They already know that mRNA drug or whatever likely won't cure them. It's possible some new scientific knowledge of the drugs' effects can still be gained even when it doesn't cure them.
Given the realistic odds, the primary purpose (from society's perspective) of letting of terminal patients participating in unproven drug trials would be medical research rather than "Hail Mary cures". From the patient's perspective, of course they hope it improves their health.
There's a lot of problems with it, though. Even if advancing medical research was the main goal-- ad hoc use in dying patients provides unclear data. Medical research wants clear inclusion criteria, metrics, reduced statistical noise from similar patients, predeclared outcome measures, and economies of scale in running trials.
We already let terminal patients participate in medical research, but only inviting patients at times and meeting criteria that optimizes the research. Also, only where IRBs have found it to be ethical-- this means not replacing a drug with proven benefit with an experimental treatment with unknown benefit recklessly.
But terminally-ill patients push for inclusion beyond this: to be dosed when there is not an active trial, or to be dosed when they are so sick that the outcome would be difficult to interpret or compare, etc. This isn't for medical research, this is for self-preservation.
>There's a lot of problems with it, though. [...] Medical research wants clear inclusion criteria, [...] , but only inviting patients at times and meeting criteria that optimizes the research [...] But terminally-ill patients push for inclusion beyond this: to be dosed when there is not an active trial, [...]
Your objections are sensible but that's beyond the scope of this author's argument. I'm just taking author's following comment at face value: ">The FDA was loathe to approve initial mRNA human trials, even when those trials would have been full of people like me: those who are facing death sentences anyway."
If it's the drug researchers who don't want the author as a test subject, that's understandable. But he's arguing the _FDA_ shouldn't be the one blocking his participation.
For the situations where there's "no active trial" caused by the FDA not greenlighting the experiments, then of course, the complaint will be that "terminally ill patients want to participate in trials that don't exist". That's sort of a circular argument -- caused by the FDA.
Again, if there's no active trial because the medical researchers themselves are not yet ready, that's understandable. But if the drug scientists are ready but the FDA is not, that's a different issue and it's the focus of the author's essay.
All that said, I don't know how much scientific progress the FDA holds back because of "safety". I'm just trying to explain the author's position.
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> we’ll know that we at least we made significant effort, and gave everyone a better chance, rather than settle for a “Hail Mary” drug near the end.
I think this is a sensible take for someone who is not facing their own imminent mortality. But no matter what we do, the game theory of terminal diagnoses means there will always be incentive to go for a Hail Mary in the end.
>How often do these drugs, etc turn out to effective?
This is a big point a lot of people ignore. I feel terrible for the author, but if there hasn't been a single drug invented so far that has any real chance of curing him, it's extremely unlikely that this one out of reach drug is going to be the drug that can save his life. I don't really know much about medicine, but I'm pretty sure that progress is usually incremental as well, so you start out with drugs that cure some patients and work from there instead of having some massive breakthrough.
Just "invest more"?
Who does the investing? Not "The Market". The incentive isn't to cure but to treat. The Market wants (repeat) customers.
The government? At least, in the US, we've been dismantling the government's ability to do this since at least Reagan. Even our so-called Liberals aren't pushing hard here. You'd have to look to the "fringe" Progressives within the Democratic Party to even come close.
And why? Because "fuck you, I've got mine" may as well be the national motto of the USA at this point.
People will bristle at this, certainly. Look at Biden: he's done so much!
Biden is no FDR. We need a real social safety net in this country. And, yes, we need a "right to try", even if it means waiving malpractice rights in those cases.
> The incentive isn't to cure but to treat. The Market wants (repeat) customers.
I am fed up with this argument.
Compared to a recurring treatment, a cure will:
- destroy the competing recurring treatment
- sell particularly well initially, when you still have the patent, potentially at a high price
- not stop the influx of repeat customers, since as long as people are alive, they will need medicine, and people who are now healthy because you cured them will be more likely to have a well paying job that will pay for expensive drugs
If that argument was true, no one would do vaccines, especially considering that most vaccines are not particularly expensive.
Gilead’s hepatitis cure is a real example of this[1], so it’s not some hypothetical. The sales plummeted and wall street dinged them for it.
[1] https://www.investors.com/news/technology/can-gilead-withsta...
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Agree. I can speak for oncology where the desire is always curative intent treatments. Unfortunately it’s often not possible so we settle for palliative therapy (presumably what GP meant by recurring).
Personally/professionally I think this argument comes from non-experts failing to understand the disease process and therapeutic challenges. I’ve seen no evidence or suggestion in my years of clinical practice that this assertion has a shred of truth behind it.
> If that argument was true, no one would do vaccines, especially considering that most vaccines are not particularly expensive.
While I agree with you this statement does not prove your argument. Vaccines aren’t a good example of funding for curative intent treatments.
The economics behind vaccinations are different as you vaccinate orders of magnitude more patients than would ever get the disease so even if the nefarious assumption is valid the economics may still favor this path.
Vaccines aren't a cure. They require repeat customers. You're making my argument for me.
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Indeed, there's the perverse insensitive to create a repeat customer.
Funnily, an insurance company would push against that, for a final cure. They would like to avoid to pay for repeat treatments.
But the medical insurance in the US is more of a payment scheme, and apparently is not interested in paying less. I bet there is some regulatory loophole, or a government guarantee, that makes it more profitable.
People are, perhaps understandably, very eager to advance at a faster pace, always a faster pace. Nowhere is that more evident than in the case of people facing death regardless, but in addition to the issues you raised (early failures can lead to even worse delays) there are other ethical concerns. Terminally ill people are often understandably desperate, and I would be too in their position, no doubt about it. Their families are desperate, often their doctors are desperate. That doesn't mean that their remaining life doesn't have value though, or that cutting it weeks, months or years short isn't a problem.
I think that's often lost on people, and in a more frightening way, people who are all-to-eager to use the terminally ill as ultimately disposable test subjects for unproven treatments. If it was JUST down to safety then I could see the point to an extent, but as you said efficacy is a big open question too. Of course if you're facing death then you may well want to roll the dice, but the question there too is how well you really understand the odds. The understandable implication in a lot of the discussions around this issue is that if only regulators were a bit faster, people like the author wouldn't die.
The reality is grim though, most of these experimental drugs and therapies are not effective, regardless of whether or not they're safe.
We let people have assisted-suicide for reasons from homelessness to hearing loss, twenty thousand people a year. Why do we respect their self-ownership when they're trying to die but not when they're trying not to die? Why do we get to decide what is and isn't worth the risk?
> Why do we get to decide what is and isn't worth the risk?
It's not their risk to take. Getting a drug to market is a complex process that can be derailed by any number of mistakes ranging from the mundane like improper blood draw during clinical trials to the tragic like accidental deaths that puts public pressure on the FDA to shut it down or spook away investors. Any hiccup in this process can kill it and once a patent expires, the chances of someone bringing that drug to market is almost zero, regardless of its efficacy.
The vast majority of drugs and therapies are developed via private investment and many companies go public before they make even a cent of revenue to fund the clinical trials. Public sentiment is the driving force behind their stock prices because they're literally forbidden from making money until the FDA approves their drug. Lots of them go bankrupt every year before finishing clinical trials or releasing a product and rushing the process is quick ticket there.
Patients don't get to put at risk the decades of good a drug can do because they're desperate and afraid of dying.
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Because they're trying to die, they're literally getting what they want, guaranteed. Having said that I don't agree with assisted suicide for the reasons you listed. Either way it's not as though people seeking death are being mislead as to the odds, are engaging in desperate reasoning that they'll be the lucky ones who get to live a bit longer.
You offer someone death, they get death.
This is about people desperate for cures and treatments that probably won't work, and may well give them the opposite outcome from what they hope for.
There is a difference in kind.
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3 years ago we wanted to rush the COVID vaccine and it probably saved millions.
I don't understand why we can't let people make risk reward judgments. Yes probably more people will die, but they were not forced.
I agree. Dying in anguish at the hands of your own government is far worse than dying having tried everything you could.
Did you never experience the situation where someone you care about is making a choice you think is wrong, and you want to protect them from themselves?
I'm not saying this is the right thing to do. I'm just trying to explain how it happens. People don't make good risk judgements in general, and other people think that it is good to do good by force. This is why seat belts are mandatory, smoking is discouraged, drugs are illegal, public companies traded on exchanges are strongly regulated, and so on and so forth.
> Yes probably more people will die
You really don't understand why we choose the option where less people die? We value life, I guess.
I agree with you but I also understand the argument that being given the choice between certain death and uncertain death is a form of coercion
And I'm sure that those people dying are saying to themselves "well at least I wasn't coerced"
The COVID vaccine already had an existing model to work from that was already ready to go and approved in the SARS-COV-1 vaccine nearly a decade earlier.