Medicare will no longer provide coverage of the new Alzheimer’s drug Aduhelm (aducanumab) except for people who are enrolled in clinical trials of the drug, according to a statement issued by the Centers for Medicare and Medicaid Services (CMS). The decision finalizes a policy proposal first announced by CMS in January.
This is an unusual move by the U.S. government health plan, as it’s the first time the agency has limited Medicare beneficiaries’ access to a drug approved by the U.S. Food and Drug Administration (FDA), at least for the approved medical conditions, according to a New York Times report.
Alzheimer’s disease affects about six million Americans, according to the National Institute on Aging, and most of them are age 65 or older, the age when people are eligible for the government insurance plan. It’s estimated that more than four out of five people who might be prescribed Aduhelm are enrolled in Medicare.
Aduhelm’s Fast-Tracked Approval Has Been Controversial
The approval process for Aduhelm has generated significant controversy. The drug was given the green light in June 2021 under the accelerated approval pathway, an approach that critics (including many members of the FDA advisory committee) believed was a workaround because the clinical trial data wasn’t good enough for regular approval.
Only one of the drug’s late-stage trials showed that it helped slow cognitive decline. Usually two trials showing efficacy are required by the FDA.
The accelerated approval program allows for authorization of unproven drugs for serious diseases with few treatment options if the drug has a mechanism of action that is “reasonably likely to predict clinical benefit.”
According to the FDA, Aduhelm works by reducing beta-amyloid plaques; reducing these plaques is believed to be likely to lead to a reduction in clinical decline because of Alzheimer’s disease. Per the labeling, treatment with the drug should be started only in people with mild cognitive impairment or the mild dementia stage of the disease.
Medicare Declined Coverage Because of Safety Risks and Lack of Efficacy
The lack of evidence on Aduhelm’s efficacy as well as the risk of potential harm, including headaches, dizziness, falls, and potentially serious complications such as brain bleeds, drove CMS to review the coverage policy, according to an agency statement in January.
During the review process, CMS considered more than 10,000 public comments and information from more than 250 documents, including data from a phase 3 Aduhelm trial published on March 18, 2022, in the Journal of Prevention of Alzheimer’s Disease.
While many patients and doctors submitted letters stating that access shouldn’t be limited once new drugs are approved, others argued that the drug should never have been approved in the first place.
Aduhelm’s price, which was cut in December to $28,200 from $56,000 a year, has also been cited as a concern, as some experts feared it would strain Medicare’s budget.
Ultimately, the agency decided the potential risks outweighed the benefits. "There is the potential for promise with this treatment; however, there is not currently enough evidence demonstrating improved health outcomes to say that it is reasonable and necessary for people with Medicare, which is a key consideration for CMS when making national coverage determinations,” said Lee Fleisher, MD, CMS's chief medical officer and the director of the Center for Clinical Standards and Quality, in the statement.
Patient Groups Disappointed in Medicare’s Coverage Decision
Patient-advocacy groups have expressed their disappointment about the decision. Limiting coverage to people in clinical trials is “an unnecessary and never before imposed barrier to access an FDA-approved treatment,” the Alzheimer’s Association said in a statement.
“Patients with Alzheimer's, a fatal disease, should have FDA-approved treatments covered by Medicare just as those facing other diseases do," said Harry Johns, the Alzheimer's Association's chief executive officer.
Could Medicare’s Verdict Impact Future Alzheimer’s Research?
There are three other plaque-clearing Alzheimer’s drugs in late-stage development. According to the CMS statement, any drugs approved by the standard FDA process (and not the accelerated process) that show clear patient benefit would not need to undergo further clinical trial requirements to be covered by Medicare.
Still, advocacy groups are concerned that the decision will discourage future Alzheimer’s and dementia research and innovation. CMS's decision not to cover the monoclonal-antibodies-targeting amyloid drugs, which were approved via accelerated pathway, is broad overreach, stated the Alzheimer’s Association. “Accelerated approval is a pathway created by Congress and utilized by FDA to allow for earlier approval of drugs that treat serious conditions, and that fill an unmet medical need,” the association said in its statement.
The Future of Aduhelm Is Uncertain
It’s unclear what comes next for Aduhelm. Biogen, the pharmaceutical company that manufactures the drug, is “carefully considering its options” as it assesses the business impact of the decision, according to Reuters.
The future of the drug may depend on a new clinical trial that is just beginning to enroll patients, according to an NPR report. The research could take years to complete, but Medicare could reverse its decisions if the results from that trial are positive.