CMS on Monday announced that Medicare will only pay for Aduhelm, a controversial Alzheimer's drug developed by Biogen, only for those beneficiaries who are enrolled in qualifying clinical trials.

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Background

Biogen received conditional FDA approval for Aduhelm last June, after a company analysis of clinical trial data found that a high dose of the medication provided a small benefit in slowing cognitive decline, and that the drug is effective at removing beta-amyloid proteins in the brain.

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In approving Aduhelm, FDA went against the recommendation of its independent expert panel, which had determined there was not enough evidence that Aduhelm actually improves cognitive function. The conditional approval requires Aduhelm to conduct future clinical trials to demonstrate the drug's benefits.

Since Aduhelm's approval, critics have raised several concerns over the drug, including uncertainty about whether it truly works, potentially serious side effects, and the financial burden of paying for the drug on Medicare, commercial insurers, and patients.

In December 2021, Biogen announced that Aduhelm's annual price would decrease from $56,000 to $28,200 in an attempt to "facilitate patient access" to the drug, according to Biogen CEO Michel Vounatsos.

How Medicare will cover Aduhelm

In a National Coverage Determination (NCD) proposed by CMS, the agency announced that Medicare would cover FDA-approved monoclonal antibodies that target amyloid in Alzheimer's disease—which includes Aduhelm—only through "coverage with evidence development," meaning that only Medicare beneficiaries enrolled in certain randomized controlled trials can receive coverage for the drug.

CMS would review every clinical trial submitted and determine whether the trial meets the agency's criteria. Trials will be required to address whether the use of anti-amyloid antibodies in Alzheimer's produces statistically significant and clinically meaningful results in cognition decline and function.

The trials will also be required to include a diversity of patients representative of the national population of people diagnosed with Alzheimer's. Trials sponsored by both CMS and NIH will be covered under the NCD.

The final decision on the NCD is expected by April 11 following a public comment period running through mid-February.

Reaction

According to CMS Administrator Chiquita Brooks-LaSure, the NCD will give the public "a clear, trusted, evidence-based decision that is made only after a thorough analysis of public feedback on the benefits and risks of coverage for Medicare patients."

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Lee Fleisher, CMO for CMS and director of the Center for Clinical Standards, said the NCD is limited because "our obligation is to ensure that we look at approved treatments that are shown [to have] benefits and those benefits outweigh the harms."

The NCD "is the result of robust evidence analysis conducted through a thorough review process that found while there may be the potential for promise with this treatment, there is also the potential for harm to patients," Fleisher said. "This harm may range from headaches, dizziness, and falls, to other potentially serious complications such as brain bleeds."

CMS' decision is very unusual for the agency, James Chambers, a researcher at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, noted. Chambers said it's "the first time that CMS [has] limited Medicare beneficiaries' access to an FDA-approved drug in this way."

For its part, Biogen was critical of the NCD, arguing that it "denies nearly all Medicare beneficiaries" from accessing Aduhelm and other amyloid-targeting therapies.

"It is imperative to change this draft decision to be aligned with reimbursement for other therapies for progressive diseases, where patients have immediate and equal access to medicines approved by the FDA," the company said in a release.

The Alzheimer's Association said the NCD is "a shocking discrimination against everyone with Alzheimer's disease, especially those who are disproportionately impacted by this fatal disease, including women, Blacks, and Hispanics."

However, some health policy experts argued that the CMS decision strikes an appropriate balance.

Ezekiel Emanuel, chair of the department of medical ethics and health policy at the University of Pennsylvania, said on Twitter, "The Coverage with Evidence Development designation is how Medicare should cover drugs not yet proven—with evidence! This will help us learn a lot about whether Aduhelm really works to reduce cognitive decline."

HHS Secretary says Medicare should revisit Aduhelm premiums

Meanwhile, HHS Secretary Xavier Becerra on Monday said that Medicare should revisit its premium recommendations for 2022 Medicare Part B "given the dramatic price change of the Alzheimer's drug, Aduhelm."

CMS previously had cited Aduhelm's cost as a factor in the 15% increase in Medicare Part B premiums in 2022.

"With the 50% price drop of Aduhelm on January 1, there is a compelling basis for CMS to reexamine the previous recommendation," Becerra said. (Commins, HealthLeaders Media, 1/12; George, MedPage Today, 1/11; Belluck, New York Times, 1/11; Herman, Axios, 1/11; Goldman, Modern Healthcare, 1/11; Goldman, Modern Healthcare, 1/10; Herman, Axios, 1/10)