Comment: Medicare right to curb access to new Alzheimer’s drug
Published 1:30 am Wednesday, April 13, 2022
By Arthur L. Caplan / Special to The Washington Post
The decision by the Centers for Medicare and Medicaid Services to decline to pay for an expensive new Alzheimer’s drug unless people who take it are part of a clinical trial was taken as a defeat by some advocates for Alzheimer’s patients. In fact, the decision represents a triumph for the scientific process. That process held firm, despite intense lobbying efforts both by the drug’s maker and by many of those advocates (who are understandably desperate for any new treatment).
I had a ringside seat on the controversy. About a week ago, I quit the scientific advisory board of the Alliance for Aging Research — as did several other members of the board — because the group was lobbying CMS to pay for the drug for nearly all patients with Alzheimer’s.
In doing so, the alliance had joined a well-organized effort that, fortunately, was turned back. The long saga that began with the controversial “accelerated” approval of Aduhelm by the Food and Drug Administration last June, has come to a reasonable conclusion. Now, instead of the government spending tens of billions of dollars annually on a treatment that may not help patients at all — and that has significant adverse effects — we will learn if the glimmer of promise Aduhelm showed in one clinical trial holds up.
When Aduhelm first came before the FDA, its independent advisory committee of scientific experts decisively recommended against approval. Two large studies had looked into the drug’s effectiveness. In both, it appeared to lower the number of beta-amyloid plaques that appear in the brains of many patients with the disease. But that’s different from showing actual effectiveness, and on that score the results were more mixed. One study found that high doses of the drug slightly slowed the soul-robbing progress of the disease in patients with early-stage Alzheimer’s. The other — also focused on early-stage patients — showed no benefit and was terminated before completion. Despite the panel of experts’ view that the data wasn’t good enough, the FDA — perhaps keenly aware that no new Alzheimer’s drug had been approved in 18 years, and that families were looking for hope — gave it a bright green light. Three of the expert panelists promptly resigned.
The FDA’s decision put the Centers for Medicare and Medicaid Services in a difficult position, because Biogen slapped a price tag of $56,000 a year on its drug. (After an outcry, it cut the figure to $28,000 annually.) And patients would probably be getting it via infusions for the rest of their lives. Expensive brain imaging and blood tests would also be required regularly for patients on Aduhelm, because the drug caused brain swelling and brain bleeding (usually mild but not always) in a minority of patients.
Many academic centers that treat Alzheimer’s patients concluded that, the FDA’s decision aside, the drug was not ready for general use, and they wouldn’t prescribe it. But if CMS agreed to pay for it, other medical institutions would certainly have supplied it. The bill would have been staggering: 6.2 million Americans have the disease, and the number grows every year. Nearly all of those patients are eligible for Medicare. A conservative estimate for the combined cost of the drug, its administration and related tests and scans would be $50,000 per person per year, which would translate into hundreds of billions of federal dollars annually. CMS held the line. In January, it said it was likely to approve payment for the drug only for patients enrolled in clinical studies. Then, on Thursday, it said that decision was final.
By all means, when an effective drug emerges to combat Alzheimer’s, CMS should figure out how to fund it. But Aduhelm has not yet proved itself.
Battles over drugs and treatments of questionable efficacy and safety are becoming a permanent part of Washington life. The FDA is clearly feeling pressure from patient groups, drug companies and members of Congress to speed up approval of — and access to — drugs to treat a host of ailments, including sickle cell, diabetes, hemophilia, ALS, drug addiction and various cancers. Payers, government and private, will push back if they think the FDA is approving drugs that aren’t proved to work.
In the era of emergency use authorizations, online testimonials promoted by patient groups and the expectations established by Operation Warp Speed, asking for strong evidence before stocking new agents in pharmacies can seem cruel, obstinate or outmoded. But unless we are willing to let hope and lobbying drive access to new drugs, we need to recommit to demanding strong evidence for approval. Otherwise, we will end up paying fortunes for ballyhoo.
Arthur L. Caplan is the director of the Division of Medical Ethics at NYU Langone Medical Center’s Department of Population Health.
