The question I have is:
How does FDA approved 'compassionate use' differ from single patient 'compassionate use'?
As you can see from Magnum's efforts to get 'single patient' last year
the stumbling block was not the FDA, but rather the fact that Vertex was not allowed to charge for meds used under this exemption. Thus for every 'c u', they lost a paying customer. Hence, tons of red tape and no actual exemptions ever granted.
Would FDA approved 'c u' allow them to charge and, if so, how would they set price? Would there be grants or other funding involved?
It sounds as if, by the time all these hoops are jumped through, the drug will be approved anyway.