Washington, D.C.—Several major players in the healthcare market have declined to add the FDA-approved Alzheimer’s drug aducanumab (Aduhelm) to their national formularies.

One of the most recent was the Veterans Health Administration, even though special pricing had been promised for veterans. The VA’s Pharmacy Benefits Management group explained the decision based on “the lack of evidence of a robust and meaningful clinical benefit and the known safety signal.” Department of Veteran’s Affairs policy still will allow providers to request coverage on an individual basis for patients whose MRI scans confirm the presence of beta-amyloid plaques. Patients must be tested to ensure they do not have a gene associated with brain swelling in clinical trials and do not have a history of bleeding disorders and are not on blood thinners.

Other health systems limiting coverage include the Cleveland Clinic, Mount Sinai, and Blue Cross Blue Shield affiliates in New York, Pennsylvania, Florida, Michigan, North Carolina, and Kansas which noted that “a clinical benefit has not been established.” In making their decisions, those groups also point to significant safety concerns, and the drug’s high cost at $56,000 per year.

The Centers for Medicaid and Medicare Services (CMS) usually covers drugs that receive FDA approval, but in the case of Aduhelm, it has decided to undertake a national coverage determination process, which it projects will not be completed until April 2022. While regional Medicare contractors could set their own policies before the national decision is made, most appear to have decided to wait for the national decision. Whatever CMS decides could affect other monoclonal antibodies in the pipeline to treat Alzheimer’s, including donanemab and gantenerumab, two other beta amyloid-targeting therapies. 

CMS also could restrict coverage to particular types of patients, such as those with evidence of amyloid plaques, require repeated monitoring scans, or limit use to trials, among other options. Because about 85% of patients with Alzheimer’s disease, including most veterans, have Medicare coverage, the cost to the agency could be huge. The Kaiser Family Foundation estimates that use in eligible patients with mild dementia could cost CMS as much as $29  billion, about the cost of all other drugs CMS currently covers combined. 

The drug received accelerated FDA approval in June despite the nearly unanimous rejection of the manufacturer’s application by the agency’s Independent Peripheral and Central Nervous System Drugs Advisory Committee. As part of the approval, the FDA required a confirmatory, phase IV, trial to show clinical benefit, although the manufacturer has nine years to complete that trial.

Three advisory committee members resigned following the FDA’s approval. One of them, Aaron Kesselheim, MD, JD, MPH, a professor at Harvard Medical School and director of the program on regulation, therapeutics, and law at Brigham and Women’s Hospital, calling it the “probably the worst drug approval in recent US history,” in his resignation letter.

Three weeks after approval, following intense criticism, the FDA added restrictions to the indication that limited it to use in patients who have mild cognitive impairment or mild dementia. Unlike the clinical trials, the FDA did not exclude patients who lack proof of amyloid plaques.

In mid-July, FDA Acting Director Janet Woodcock, MD, called for an investigation by the Department of Health and Human Services’ Office of the Inspector General (OIG) into possible irregularities in the drug’s approval process and communications between the agency and aducanumab’s manufacturer. Several members of Congress have also called for an investigation. The OIG investigation is not expected to wrap up until 2023.

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