A Travesty at the FDA

Update 2021-08-20 since this is getting more attention:

Shortly after I wrote this post, the FDA backtracked a bit, and restricted the indication to only early-stage Alzheimer’s patients. This means that it’s unlikely aducanumab will cost $100 billion per year. Still, it could be extremely expensive.

Yesterday the FDA approved aducanumab, an anti-amyloid antibody developed by Biogen, for the treatment of Alzheimer’s disease. This was based on post hoc interpretation of clinical trials that were stopped early due to futility, and against the strong recommendation of the FDA’s own advisory committee. Aducanumab will be priced at $56,000 per patient per year. Since Alzheimer’s patients are usually covered by Medicare, this cost will be paid by the American public. I estimate that it could be over $100 billion per year.1 This is excluding indirect costs, such as those of monitoring for brain swelling that is a known effect of this drug.2

Putting this in context, for $56,000/year you could hire a graduate student or postdoc to work on Alzheimer’s research full-time. Imagine the opportunity cost!

Writing at In the Pipeline, Derek Lowe called this a tragedy.3 I want to go further. This is not just a tragedy, but a travesty. The FDA has become a sadistically distorted mockery of what medical regulation should look like. There can be no excuses for the level of statistical incompetence required to approve aducanumab based on the flimsy efficacy data from the trials.4

Besides the costs of aducanumab itself, we should also consider the fact that other companies will take notice at how much money Biogen rakes in. I expect lots of statistically shoddy submissions to the FDA in coming years, many of which will be approved.

Defenders of the FDA’s decision must also reckon with the fact that the FDA has still not given full approval to the COVID-19 vaccines, which have much better data about safety and efficacy. If all the lengthy advisory committee meetings were overruled for aducanumab, what’s the point of having them at all? An FDA that approves aducanumab, but not COVID-19 vaccines, is in dire need of a shake-up.

1

At $56,000 per year, 1.8 million patients could be treated yearly for $100 billion. There would be ~6 million people eligible in the United States, so it could be even more than this.

2

I don’t say “side effect”, because it might well be the only effect.

4

In a high-dose subgroup, in only one of the two trials, there was a 0.4-point difference in cognitive decline out of a 3-point scale. Plus, the FDA approved this for all Alzheimer’s patients, not only the early-stage patients who took part in the trials.