I feel somewhat closer to this person because I was once in his position as a child fighting a neuroblastoma in 1981 and some how my mother found a way to get me on an experimental list which saved my life. Half of the kids that took the drug died by the time they were teens due to heart failure, obviously I wasn't one of them, but am thankful for the chance to live a full life.
When the odds are against survival, there shouldn't be any rules limiting treatment, if it means even a slightly higher chance of winning. That's something our government has forgotten about or doesn't care about. If I was in this persons shoes, I would make it as personal as possible against those in control of the FDA. Show up at their houses, work, kids soccer practice, get in their face and let them see what their inaction is doing in person.
> When the odds are against survival, there shouldn't be any rules limiting treatment, if it means even a slightly higher chance of winning.
I agree with you. But the FDA would ask you to rigorously define "slightly higher chance of winning" -- and prove it. In the event you cannot do this, they believe that letting you try an experimental drug would be more unethical than letting you die of neglect.
So I'd rephrase that statement of yours: "When the odds are against survival, there shouldn't be any rules limiting treatment, regardless of type of treatment or odds of efficacy." ("When the odds are against survival" can be quantified, e.g. a 90% chance of dying within a year.)
I am not yet done thinking on this area, so my thoughts here are incomplete, but thay said:
Would this not also open the door for any snakeoil salesman to prey on what might be medically "hopeless" cases?
If I am ever diagnosed with something currently uncurable, but there are experimental stuff in the works, of course I'd like a shot at those experimental drugs, but I hope that me/my family would also not squander whatever assets we have on snakeoil, in which case I'd rather my surviving family was not impoverished in the fight.
I assume that, at least partially, this is why regulations in this area were put in place to begin with.
> Would this not also open the door for any snakeoil salesman to prey on what might be medically "hopeless" cases?
If you're diagnosed with something uncurable, then by definition you have a doctor or a medical team that has performed the diagnosis and is overseeing your treatment. There is, invariably, some form of treatment; in extremis, even "here, I've booked you into a hospice where they're going to load you up on opiates" is a form of treatment.
Any experimental drugs you're administered would go through your doctor or medical team. If those drugs are transparently snake oil, they should usually be very strongly advised against. If they have a halfway plausible mechanism, doc will probably say, "go ahead, roll the dice."
In any case, the snake oil salesmen aren't preying on sick people alone -- it's the sick person, plus the professionals who are in his corner, plus family and friends, etc. I don't think it would necessarily be trivial to make lots of money peddling something known to have no efficacy.
On a much more general philosophical note, there was much debate in Ancient China between Legalists, who viewed humanity as inherently evil, and Confucianists, who viewed humanity as inherently good. Where you come down in this debate seems to depends on that view. If you believe that men are inherently evil wreckers, you need stringent and indeed draconian regulations to keep them in place. If you believe that men are good, and that lives saved are on balance the greater aim, you should argue against draconian regulations and limitations on treatments.
The obvious case is that some are evil and some are good. And that's enough for the evil people to find the good people. (This is how Internet-connected computers secure works / fails.)
Before anyone shows up at FDA decision makers' kids soccer practice please consider things from the point of view of an FDA decision maker. They have to make a choice which they know will lead to both type 1 and type 2 errors. For life saving medicine, when the decision isn't clear, both type 1 and type 2 errors are awful. It's likely they already feel the burden of responsibility.
When the odds are against survival, there shouldn't be any rules limiting treatment, if it means even a slightly higher chance of winning. That's something our government has forgotten about or doesn't care about. If I was in this persons shoes, I would make it as personal as possible against those in control of the FDA. Show up at their houses, work, kids soccer practice, get in their face and let them see what their inaction is doing in person.