Funding is an issue for all “orphan” diseases and syndromes.
The FDA committee makes a “cost versus benefit” decision implicitly and/or explicitly with all of its decisions.
AIDS activists, notably the recently deceased activist Spencer Cox, made their name by beating up FDA to waive the normal rules and approve half-baked drugs given the high death rates back in the 1980S.
CFS activists have been trying the same argument with the FDA based on the severe debilitation of CFS, in some cases leading to suicide. “A marginal drug is better than no drug” is the argument.
If FDA approved Ampligen, the hard-pressed US health “insurance” system, especially under Obamacare, might be required to reimburse the $25,000/year Ampligan treatment, as it has with expensive MS infusion drugs, for example. Cost IS a consideration in drug approvals.
In the case of the MS infusion drugs, the benefits were much clearer and the patients that would benefit could be identified with greater accuracy than CFS (no accuracy actually, in CFS it seems). So Ampligen failed both the cost and benefit sides of the cost/benefit analysis, except for the four members of the panel who still thought it did!
Why this intriguing if ill-proven medication would need that sort of expense to crank out of the labs, when it had been discovered in the sixties, is unknown to me. Not all orphan drugs do. The manufacturer is going to want something to recoup their troubles, of course. Whether that part spells the lion’s share of the quoted $25 grand, I do not know. That doesn’t necessarily excuse a poor test, because issues such as the majority of the FDA commissioners alleged are not esoteric things to anyone versed in the science.
I’ve long had a quasi-libertarian view on medicaments of any sort. There could be two classes of medications, that which has been officially blessed by the bureaucrats, and that which has not been. If I were king, the second class could still be administered by doctors, as long as they participated in a sufficiently large liability insurance plan against harm caused by what they administered. Market economics, rather than politics, would then dictate what other drugs would be tolerated in the society.