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Comment by patrickg_zill | Hacker News Reader

There surely has to be a middle ground in some cases between the two cases of $100 million trials and essentially unregulated and unaccountable hotel room doctors.

Could there be a series of exams that a would be patient would have to write, to demonstrate that they truly understand the risks, and what protocol is to be followed?

In investment they have an "accredited investor" category to avoid higher risk investments from being offered to those who can't afford the risk.

If you have a chronic disease and pass the exam then you become an "accredited trial patient".

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You can sign up for clinical trials already. What you propose sounds like clinical-trial-but-not-really, at the end of which it is unclear if the new compound is safe and efficaceous,

Efficacy is a bigger problem than safety. Think about all the products you see advertised on a typical day (especially if you watch TV). How many of thing actually work? Anti-oxidants, probiotics, shampoo with gobbledygook ingredients in it, homeopathic teething ointment.

For the most part, all this is just a waste of time and money. But for medicine, things that don't work create an opportunity cost--someone uses an anti-oxidant-packed cancer treatment and dies. It's a basic information asymmetry that exists in the market: its too hard to prove that things work, and the wonders of modern marketing ensures that lots of things that don't work still sell like crazy.

Efficacy tests address that problem. But proving efficacy is hard and expensive. But they're probably not "too expensive." That is to say, if you made clinical trials cheaper by just testing for safety, you'd probably suffer a net economic loss due to people spending money and dying from products that don't work than you would on the trials.

Proper medical trials don't guarantee efficacy either. For example denavir or abreva. FDA approved yet utterly useless.

So why are they approved? Because there's nothing better. It's the same here, make these trials easier for conditions that otherwise have no good options.

Isn’t that what the “compassionate use” exemption is for?

Unfortunately there is a middle ground, made possible by the FDA's toothless regulatory regime. It isn't benefiting patients or advancing scientific progress.

https://en.wikipedia.org/wiki/Burzynski_Clinic

Trump wanted to introduce “right to try”, which I think is a great idea. Got shut down in Congress. Don’t recall why.

IIRC, part of the argument against is that you're allowing biotech/pharma companies to test on vulnerable populations. Here's a story on NPR about the pros and cons: https://www.npr.org/sections/health-shots/2017/03/03/5177969...

Sure, that’s inextricably a part of the deal. Don’t sign on the dotted line if you’re uneasy about it. As long as nobody is forcing you to do it, I don’t have a problem. The situation where I can try things (at my discretion) is strictly better than the one where i unconditionally die.

>Sure, that’s inextricably a part of the deal. Don’t sign on the dotted line if you’re uneasy about it.

Well that does sound like a good reason for Congress not to sign on the dotted line, given the definition of 'it'.

The information asymmetry is a real problem.

Is it more of a problem than definitely dying, though? That’s the real question here.

1. You unconditionally die in extreme agony.

2. You weren't dying before, but you are now.

3. Not dying, but the extreme agony is back.

4. Still not dying and no pain, but you now look like one of the aliens from Close Encounters. Career choices are limited.

...

That refers to access to drugs that have passed Phase 1 safety trials. The study here wasn't close to that.

Many reasons

- there are plenty of quacks who already con people - people who do actually believe their product works, but it doesn’t - things that may work but also may kill you (remember that drug trial in London a few years ago that had got through all pre-human trials without a problem?) - placebo is real, and the pre-approval access ideas generally are a pay-to-play concept, and I’m not sure people would want to pay for a blinded treatment - people talking about these trials always seem to say “once basic safety tests are done”, but phase 1 and 2 are the basic safety tests. Phase 3 require enough people that drug companies are often actively working to find participants.

The real problem here is that universities etc keep posting hyperbolic articles saying “we have found something that may cure X”, with the rider for studies and fda approval are necessary.

Laypeople interpret that as meaning the fda approval is preventing them from accepting patients for approval, when typically the component in question is not remotely close to a treatment:

- it’s just binding to a target invitro. -There’s no information on what happens one it has bound. - there’s no evidence it can even get to the target invivo anyway

- animal trials are only advisory. There have been many super effective drugs and vaccines that have no effect in humans

- can the get past the immune system? Will the immune response kill a person

- I’ve seen articles about compounds that would (and turn out to be) toxic

Etc etc

But all a patient sees is the “may cure X” bit and so demands a right to use the drug.

That’s why these things are a bad idea

> that drug trial in London a few years ago that had got through all pre-human trials without a problem

I asusme you mean the TGN1412 trial [1,2].

[1]: https://en.wikipedia.org/wiki/Theralizumab

[2]: https://www.nejm.org/doi/full/10.1056/NEJMoa063842

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