On June 7, 2021, the U.S. Food and Drug Administration (FDA) approved Aduhelm (aducanumab), a new Alzheimer’s drug for the treatment of the disease. Aduhelm was approved using the “accelerated approval pathway,” which can be used for a drug for a serious or life-threatening illness that provides a therapeutic advantage over existing treatment. According to the FDA, “accelerated approval can be based on the drug’s effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients, with a required post-approval trial to verify that the drug provides the expected clinical benefit.”
Was the approval of Aduhelm the breakthrough treatment long sought to combat the devastating effects of Alzheimer’s disease? Well, it depends on who you speak with. Proponents of Aduhelm will argue it is the first new treatment approved for Alzheimer’s since 2003 and is the first therapy that targets the fundamental pathophysiology of the disease. Opponents of the FDA approval, which includes at least three FDA Committee members who resigned over Aduhelm’s approval, will point out the clinical trials to date have proved inconclusive, and there is “no compelling, substantial evidence of treatment effect or disease slowing.” One FDA advisor who resigned in protest called the decision, “probably the worst drug-approval decision in recent US history.”
Now, the issue turns to a practical issue: How is our health delivery system going to handle this new Alzheimer’s drug that millions of Americans will potentially want access to? The drug will cost $56,000 per year. The drug is administered through monthly infusions which our health care is not established to handle. Will Medicare pay for this new Alzheimer’s drug? I have already been told by one prominent dementia expert that their practice will require a PET scan as a prerequisite for prescribing the medication. A PET scan is not currently covered by Medicare, and the out-of-pocket costs can be $5,000 or more.
Recently, I had a discussion with a client’s son who is a geriatrician regarding his mother with Alzheimer’s disease. I asked him, “if this medication was available while your mother was still alive, would you give your mother the drug based upon what you know today?” The answer was unequivocally “yes,” he said.
Thus, in reality, the FDA approval decision was based as much on hope as on science. Aduhelm may represent hope for Alzheimer’s patients and their families who have waited years for new treatments to come along. However, that hope is likely to come at a high cost to Medicare, beneficiaries, and taxpayers.