Yesterday the Food and Drug Administration authorized Biogen to market Aduhelm, the first anti-Alzheimer’s Disease drug it has approved in 20 years. This monoclonal antibody has been shown to reduce the amyloid plaque that accumulates in the brains of patients with Alzheimer’s disease. While still controversial, most researchers believe the build-up of this plaque is somehow responsible for the progression of dementia in these patients. The FDA approval came despite the fact that its advisory panel recommended against it, contending that the clinical data did not convince them that the drug is efficacious. But the FDA approved the drug under its “accelerated approval pathway,” created to help address the problem of drug lag, where many patients suffer or even die waiting for permission to see if a drug that may be helping patients in other countries can be used by them. Using this pathway, the FDA concluded that the drug is reasonably likely to yield a clinical benefit.

The approval was criticized not only by members of its advisory panel, one of whom resigned from the panel in protest, but by many clinicians who treat Alzheimer’s Disease patients. Dr. Robert Howard of the University College London told MedPage Today:

Regrettably, the FDA has ignored high quality scientific evidence of non-efficacy provided by the large and carefully conducted phase III studies. They’ve effectively approved an expensive placebo with unpleasant side effects on the basis of action against brain amyloid levels, an action that has already been shown to have little or no effect on cognitive and functional decline with this and earlier agents. Happily, science generally finds a way of correcting itself when it goes astray. It’s too early to know how this will happen with aducanumab [Aduhelm].

Bloomberg columnist Max Nisen complains that this new drug will cost Medicare $56,000 per patient per year for what many experts believe is an unproven drug treatment. That’s not the fault of the FDA drug approval process, but rather the fact that Medicare is legally prohibited from negotiating drug prices and must cover most FDA-approved drugs.

The controversy over the FDA’s approval of Aduhelm is another example of why the FDA should not be basing the approval process on its determination of a drug’s efficacy. That falls under the realm of real-world clinical research. Prior to 1962, drug makers were required to convince the FDA their product was safe to consume and met the FDA’s criteria for providing product information, use, and dosage on their labels. But the 1962 Kefauver‐​Harris Amendments to the Food Drug and Cosmetic Act of 1938 added the additional burden of proving the drug’s efficacy in treating the condition for which it was developed. Efficacy requirements add years to the approval process.

Ironically, once a drug is approved for the treatment of the condition for which it was initially intended, the FDA places no restrictions on using the drug in any other setting. Using it to treat a condition for which it was not initially approved is called “off‐​label,” because the label is only allowed to state the condition for which its use was FDA‐​approved. An estimated 20 percent of prescriptions are for off-label use.

It is reasonable to wonder why, after doctors wait several years to get permission from the FDA to treat their patients with a drug for condition “A,” the FDA in principle trusts doctors to use their clinical judgment, based on their knowledge, experience, and analysis of real-world trials, to treat conditions “B thru Z.” Why not skip the efficacy component of the approval process and speed things up?

As Michael Cannon and I wrote in our white paper, dissatisfaction with the FDA drug approval process spurred the “Right to Try” movement, culminating in the national Right to Try Act in 2018.

Regardless of the FDA approval, the likelihood is that clinicians will share and rely upon real-world trials before deciding to recommend this drug to their patients. But that’s what always happens and how it should be. The FDA should stick to evaluating a drug’s safety and stop wasting the precious time of patients by running its own efficacy studies.