As you probably know, the FDA recently approved a monoclonal antibody called aducanumab as a treatment for Alzheimer's disease. To make a long story short, there's no good evidence that it actually has any effect on dementia; the approval is based on its effectiveness in removing abnormal plaques of a protein called beta-amyloid from the brain. These are associated with Alzheimer's disease, but there's no evidence that they cause it, or that removing them improves symptoms. Aducanumab can also have some very troubling adverse effects such as brain swelling. Oh yeah -- Biogen plans to charge $56,000/year for the treatment.
Now, if you don't think this makes a whole lot of sense, you'll be even more nonplussed to learn that it's actually quite common.
The FDA approved aducanumab through its “accelerated pathway,” a process created in 1992 to hasten approval of “drugs that treat serious conditions, and that fill an unmet medical need.” Such approvals are based on surrogate endpoints, which the agency defines as “a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit.”
Surrogate endpoints stand in contrast to direct measurements of patient outcomes, often referred to as clinical endpoints, such as living longer, symptom relief, or improved quality of life. The FDA now has multiple expedited pathways. Although accelerated approval was developed for life threatening diseases without treatment options, the agency has steadily expanded its use far beyond the original intent. In 2018, 73% of licensed drugs (43/59) received expedited approval,4 and many of those drugs treat conditions that are either non-life threatening or have existing treatments, such as gout and hypertension.
Once drugs are approved on this basis, the manufacturer is supposed to run "confirmatory trials" to show that there is indeed a clinical benefit. But they often delay doing so for years, and even when the trials fail to show benefit, the FDA often does not revoke the license. There is obviously grave harm to society when doctors continue to prescribe useless medications that generally can have adverse effects; purchasers of insurance and taxpayers continue to pay for them; and they crowd out more effective treatments.
I don't know enough about the inner workings of the FDA to say why this happens, but "regulatory capture" is a well-known phenomenon. Basically the hypothesis would be that people who work for the FDA in many cases either have had, or hope to get, jobs with drug companies; and/or they have friendly relationships with drug company employees who they work with on applications. In any case, this has to stop. I don't suppose Joe Biden is reading this, however.
2 comments:
The issue of regulators being corrupted by the purveyors of the stuff they regulate seems to be almost impossible to solve.
But I believe we have a model of how it can be done in our very midst - sports! I watch too much college football, but in doing so I've been impressed by the effort expended to make correct, and unbiased, calls during the game. A video replay of a questionable call is analyzed with lawyerly attention to the minutia of the applicable rules. It's remarkable how much effort is put into making the playing field as level as possible. Even factors that mitigate against fairness, like home field advantage, are at least acknowledged when judging the quality of a game.
A ref whose 'bad calls' continually go in favor of certain teams won't stay long in the profession, and I doubt that refs get cushy university positions as a reward for their service when they retire.
It is ironic that the average American is much more invested in, and knowledgeable about their chosen sport, which means almost nothing in the long run, than they are in the governmental units which will have a profound affect on their lives in the long run.
I wonder if a good way to get through to folks like this would be to suggest changes to their sport analogous to the underhanded moves currently being made in politics. For example, if the home team could conduct an independent audit of the game, and invalidate it if they 'discovered' too many bad calls made during it, the opposing fans would be incensed, and even the home fans would lose interest since their victory had lost credibility and become an embarrassment..
Problem is, the incentives don't run the same way. Sports teams compete but the league and it's various franchises are really one entity: an entertainment corporation bringing eyeballs on screen and asses in seats. With NDAs and other forms of regulatory capture it's more like an exhausted nanny with a brood of clever, ruthless, and indefatigable children. No matter her intentions they just keep coming up with new shit to pull and, if she cracks down, they complain to their parents and get her fired.
We need a fourth branch of government - the public censor - to chase down anything that looks even remotely like corruption.
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