The agency's recent decision to approve aducanumab offers more evidence of a broken system
by Vinay Prasad, MD, MPH, June 16, 2021
FDA's recent approval of a controversial drug -- aducanumab (Aduhelm) -- to treat people living with Alzheimer's disease shows just how broken the agency is, and reminds us that we all have to pay for it.
A series of events that has unfolded since January tells the story.
In late April, the FDA's Oncologic Drugs Advisory Committee reviewed six accelerated approvals -- a provisional pathway -- involving a group of cancer immunotherapies where clinical trials had failed to confirm that the drugs extended survival or improved quality of life. Yet, in four of the six cases, the advisory committee voted to keep the accelerated approvals intact. The lesson was painfully clear: once the toothpaste is out of the tube it is hard to get it back in. Once doctors get used to using a therapy, pulling a drug from the market or in this case, revoking indications, is hard -- even after the drugs fail to confirm benefit.
On June 7, the FDA approved aducanumab for the treatment of Alzheimer's disease. The drug received accelerated approval because it showed it could reduce the rate of amyloid plaque on scans. What remains uncertain is whether this reduction in plaque means Alzheimer's patients live longer or better lives -- and notably, the totality of the clinical trial data do not show that. Moreover, the drug has various side effects and a whopping price tag: $56,000 a year.
In response to the FDA's approval, three members of the Peripheral and Central Nervous System Drugs Advisory Committee who opposed approval of the drug, quit the panel in protest. Aaron Kesselheim, MD, JD, MPH, a Harvard professor called the drug "problematic," and argued that there was little evidence it would help patients. Writing in The Atlantic, Nicholas Bagley, JD, and Rachel Sacks, JD, MPH, estimate that if the drug is prescribed to just one-third of eligible patients, it would cost Medicare $112 billion a year -- a massive figure that dwarfs any other medication.
What Is FDA Thinking?
In the last 3 months we have seen that the FDA does not have the ability to revoke accelerated approvals, even when the drugs and their sponsors fail to meet the promises made. And to wash that down, the FDA has now approved another uncertain drug for people suffering from Alzheimer's, against the wishes of a different advisory panel. With Alzheimer's disease affecting 6 million Americans, the financial and human implications of the approval are staggering.