So, here's the thing-- drugmakers can get compassionate use exceptions.
But the pharmaceutical companies really want to prove that their drugs work. If their drug doesn't work, nothing is lost or gained by having people try it.
If the drug does work, but the study of it is confounded by giving it to people in a haphazard way, such that we don't know if it works--- more people suffer.
It sucks, but most things don't work. Occasionally people are screwed by not being able to get into a trial for something that might have saved them or lengthened their life. But much more often they're spared false hope and suffering from side effects, and we end up with trials that we can trust.
Yes, I do mean that FDA is worried about getting bad PR.
The incentives of the FDA are unfortunate. If they don't approve a drug that would have saved 100k people there is no bad press for those 100k deaths. But if they approve a drug that kills 1k people there is a lot of bad press.
So they have strong incentives to not approve anything unless absolutely needed.
