In a post back in September, I had this to say about the FDA:

The FDA would gladly see half of the American people die while they consider and reconsider for years approving some drug, in order to establish the proposition that the FDA and only the FDA has the bureaucratic say-so to determine when and how a drug can be marketed. 

And, to prove that I'm not making that up, the FDA a few days ago issued its ruling on a new drug application from a company called BioMarin for a drug called Kyndrisa intended for the treatment of Duchenne's muscular dystrophy.  The ruling is that the company has not yet submitted sufficient proof that the drug is effective to satisfy the FDA.  Tests must continue, unless the company gives up.  Oh, meanwhile there is no approved treatment whatsoever for this disease.  It arises from a genetic defect limited to boys.  About one boy in 3500 gets it, meaning about 500 new cases per year in the U.S.  It is 100% fatal.  Typically, the boy is in a wheelchair by his early teens and dead somewhere between the ages of 20 and 30.

The issue here is not that the FDA legitimately thinks this new drug is some kind of snake oil, or even that it has harms that outweigh the benefits.  The opposite.  The cause of Duchenne's has been identified (at least, within the limits of our flawed scientific processes), namely lack of a protein called dystrophin; and the drug in question (along with others under development by other companies) has been specifically designed to supply the missing protein.  Nor is the issue that no patients in trials to date seem to have benefited from the new dystrophin-supplying drugs.  For example, this from the Wall Street Journal on January 20 (relating to another one of the drugs, on which an FDA ruling is expected imminently, but anticipated to be equally negative):

Kathryn Wagner, a leading Duchenne physician at Johns Hopkins School of Medicine, says she plans to speak in favor of the Sarepta drug at the coming FDA meeting. She said she has seen patients benefit from the drug in clinical trials.      

Instead, the problem is that the disease is rare enough and serious enough that it is very difficult to get sufficient numbers of patients into the clinical trials to meet the FDA's standards of "statistical significance."  (One of the clinical trials for the drug Dr. Wagner was discussing is reported in the Journal article to have only 12 patients in each of the treatment and control groups.)  So the bureaucrat's answer is obvious: the trials will continue -- and continue, and continue, and continue -- until our standard for establishing our prerogative and maintaining our fiefdom has been met.  Meanwhile, thousands of innocent boys and young men become crippled and die unnecessarily?  What does that matter, when bureaucratic control is at stake?

Researching this post, I came across a 2014 debate sponsored by the Southwestern Law School Law & Medicine Society and the Federalist Society between libertarian NYU law professor Richard Epstein and a law/medicine guy from Southwestern named Ryan Abbott.  The subject of the debate was the closely-related subject of the FDA's unending efforts to quash speech about off-label uses of otherwise-approved drugs.  Abbott took the pro-FDA side of the debate.  Abbott's presentation gives a good sense of the progressive's reverence for the all-knowing and perfect bureaucrats -- who are always referred to as "we" because the true progressive feels himself to be one of this privileged elite.  And meanwhile, the mere non-bureaucrat is treated as ignorant, helpless and completely incapable of making any decision or acting for himself.  Excerpts:

The central problem with off-label drug use is that we have an information deficit. When the FDA approves a drug for on-label use, that approval is based on scientifically valid and statistically significant evidence that says, we are going to give you a drug, which is potentially dangerous, but it is likely that the benefits outweigh the risks. We know that because we have studied the drug in a controlled environment. That information is simply not available with off-label use.

Over 70% of off-label prescriptions used are not based on scientific evidence or significant scientific evidence.2 That’s a real problem because all drugs have a risk of serious side effects, and patients shouldn’t be exposing themselves to risk without evidence that a drug will be effective. . . .    

So not only are patients using drugs we don’t know are safe and effective, but we are not getting good feedback to inform future practice. . . .  How do we balance patient access with preventing harm, and what role should the FDA play? 

The whole approach is based on the dead-wrong idea that an elite can achieve perfect knowledge and make better decisions than individuals acting in their own interest.  Could a guy fancy enough to have both law and medicine degrees not know that all important human decisions are based on imperfect information and involve some degree of risk?  The answer is yes.  And it's not just Mr. Abbott, and not just the pooh-bahs at the FDA who keep drugs off the market while thousands die, but the entire federal bureaucracy that has bought into this narrative.