The Aduhelm Fiasco

 

In June of last year, to great media fanfare, the FDA approved the new drug Aduhelm (aducanumab) to treat Alzheimer's disease. Six million Americans suffer from Alzheimer's, which is a horrific and ultimately fatal form of dementia, and available drugs typically delay cognitive decline by only a few months at best. So any new medication is eagerly welcome. If it works, that is. And if anyone can afford it.

Two clinical trials of Aduhelm had concluded that the drug was so unlikely to slow the progression of the disease that both trials should be stopped early. They were.

Six months later, Biogen, the manufacturer of the drug, announced that they had analyzed the data more carefully and that the study group receiving the highest dose had actually improved. Based on this new information the FDA convened an external advisory committee to examine the evidence. 

"The totality of the data does not seem to provide sufficient evidence to support the efficacy of the high does," the FDA's internal analysis concluded, noting that even the "favorable" results presented by Biogen produced only a 0.4 point improvement on an 18 point scale. The advisory committee voted 10 to 0 against approving Aduhelm.

Instead, the FDA approved the drug, justifying its decision by citing that it appears to reduce the amyloid plaques often seen in Alzheimer's patients - despite the fact that there is no proof that the plaques cause the disease, rather than just being a symptom of it. Three  committee members resigned in protest. Further, the drug was approved for all Alzheimer's patients, not just the early stage patients on whom it was tested. (Did I mention that the list price of Aduhelm is $56,000 a year?)

Fortunately, new leadership at the FDA is taking a new look. In July, 2021, the FDA changed its recommendation and said that only people with early stage Alzheimer's should use the drug. That same month, the FDA's Acting Director ordered in independent investigation of the agency's approval process, citing an "off the books" meeting between the head of the FDA's neuroscience division and Biogen executives.

So there you have it. An enormously expensive drug that is mostly ineffective and has side effects such as brain bleeds and swelling approved against the advice of the FDA's own annalists and independent advisors. Doesn't sound like anything is amiss here to me. How about you?