Comment by A_D_E_P_T

There are already many "compassionate use" exceptions out there already. I've family friends be grated that. It helps the pharma company as well as the patient. I'm sure that will happen here.

Yeah putting myself in the shoes of someone with this disease or a loved one with this disease I would be so incredibly angry that we weren't allowed to try something when the alternative is certain death

Somebody has to pay for the trial. Drugs are expensive and the amount needed to dose a single person is orders of magnitude more than mice. So who funds the study?

If it's the government or philanthropic fund, you have to put in a grant and show that it's competitive in terms of preliminary results etc.

If it's the drug companies, you have to deal with lots of complicated things with combination therapies. Drug companies don't like their drug paired with other drugs that aren't in their portfolio. They also need to see a way to make a profit on it, which means they need to evaluate whether this is the most likely successful trial, assess bang for buck etc.

If you want to go compassionate use, then you need to get the pharma to donate the drugs or insurance to cover it. This is spain, so I guess insurance is just the government since they have universal healthcare. I have no idea how that works but I am guessing it doesn't move fast.

> what's the worst that can happen?

The patient dies from complications of the drug's use before the cancer.

Things are, at least, getting better with the passage of right to try laws: https://en.wikipedia.org/wiki/Right-to-try_law

These drugs seem to all be only allowed after Phase 1 trials, so still not quite at the level you're describing here.

Knowing whether drugs work isn’t trivial. Patients are typically very heterogeneous in their responses to drugs. For example, pembrolizumab (the most successful cancer drug ever) typically only works in, say 30% of patients depending of the cancer type. Just throwing therapeutic ideas out there and letting physicians sort out how to use them and in which patients, isn’t a panacea. Looking at clinical data can be like star gazing even in planned studies. Structured, statistically powered studies, and costly rigorous assays on biomarkers and correlative studies are essential for understanding how and in what patients drugs are working. I’m all for expanding access to drugs, and there is abundant waste and greed in big Pharma and venture, but there are also people doing hard expensive science, medicine and manufacturing. Im not sure I have the answer. A “yelp for medicine” won’t improve immediate outcomes, nor longer term understanding and progress. A great and excruciating read about the tension there is a real-time blog (the story’s story) that was written by Jake Seliger unt he passed in 2023.

That’s because we have a system that financially punishes fraud and mismanagement which is what we want and should keep.

There should be a way for terminal cases to volunteer for early trials and I believe there is already legislation that provides that but it’s not used and funded enough.

IMO, if you view your question from the ethical framework of "do no harm" i.e. the hippocratic oath instead of "move fast and break things", I can clearly see reason for the apprehension. The standards aren't positioned to catch "quack medicine" but to require full understanding before asking someone else to put something in their bodies. It's somewhat of an entitled stance that youd be okay with other people possibly needlessly dying in any circumstance for something experimental, and not one I'd ever want taken as an official stance by a regulated medical body.

If nothing else, it's not the only hail mary you could potentially go out on. Would you give up the potential of being part of a trial for a drug that has actually done the work and demonstrated real promise in various pre-trial tests just to take a gamble on this alternative which hasn't even had the initial test replicated yet?

Further there is the potential for a false negative. If they don't understand enough about how the drug would work in humans, they may trial inappropriate doses or delivery methods. If those don't help or make things worse, it could be mistaken for the drug being ineffective and lead to the whole line of inquiry being abandoned. Then not only do you die, but countless others are potentially harmed by an effective version not being developed.

Finally, cancer treatments aren't just for the terminal. Drugs which primarily help during the early stages by necessity need to be trialed on people who still have a chance, maybe even a decent one, going with other, well established treatment options.

We might take it too far, but the fear of quack medicine is extremely rational.

> Our society's morbid, irrational fear of quack medicine

It is not an irrational fear.

Brandolini's Law applies: "The amount of energy needed to refute bullshit is an order of magnitude bigger than that needed to produce it."

The only way to prevent quackery is to cut it off hard before it gets started.

Wakefield demonstrated the disaster that happens when you don't.

(And if you have been reading this site for very long, you know the experimental treatments are already around--we're not currently lacking for possible cancer treatments. The problem is finding the trial. Then the problem is getting people through the process and then getting them to the trial. See: "Please be dying, but not too quickly": https://bessstillman.substack.com/p/please-be-dying-but-not-...)

good news, are current FDA has many ways of fast tracking a treatment and a willingness to do so

I had a relative who died from this around 20 years ago. 50yo slim, sportive and healthy and after going to a diagnostic as she didn't feel good, she was gone within a few months .. So yeah, if there is even a slight chance it works, this should be tried and that'd save people :(

The ethics of life and death are murky at best. When is it acceptable? 6 months to live? 1 year to live? 5 years? What is the cut point? The answer is, there isn't a clear answer. Yes, that is a cop out, but it is also true. I agree with allowing people of sound mind to make informed healthcare decisions towards the end of their lives that involve high risk. but I would be wary of weakening the process too much. We should have a push-back because people in these positions are exceptionally vulnerable and therefore easy to take advantage of and, even worse, likely to be unable to defend against abuse for very long. Weakening this process would likely lead to drug companies doing what they always do, abuse their position for money. Why even go to expensive traditional trials? Just give some promising, cherry picked, results in a press release and now people are coming to you signing every waiver and paying their last penny to live next to the trial that will kill them because the drug wasn't even close to ready. I would likely feel very different if I was near death, but that is the point isn't it? Again, I'm not saying this shouldn't be an option. I just think it needs a lot of scrutiny and a high level of skepticism.

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Ethics is a topic I would never trust HN on.