CMS finalizes narrow coverage for Aduhelm, unveils second pathway for broader access to Alzheimer's drug

It's official: Despite heavyweight lobbying from pharma, patient advocacy groups and legislators, CMS stuck to its narrow national coverage determination for a controversial class of drugs for Alzheimer's disease and dementia.

The Centers for Medicare & Medicaid Services said late Thursday that monoclonal antibodies directed against amyloid plaques that are approved under the Food and Drug Administration's accelerated pathway will only be covered when administered in eligible clinical trials, which aligns with the proposal.

Just one drug in this class has earned FDA accelerated approval so far: Biogen's Aduhelm.

The agency did broaden which trials are eligible, however. In the proposed determination, CMS said it would cover these drugs when administered in specific trials it approves. In the final version, it instead says it will cover the Alzheimer's therapies when administered in FDA drug application trials or studies conducted by the National Institutes of Health.

CMS has also added a second coverage pathway. Should a therapy in this class secure traditional FDA approval, the agency will cover it more broadly in a number of approved settings, such as data collected through patient registries or in routine clinical practice.

Any Medicare beneficiary in one of these studies who meets the label criteria for the drug would be eligible for coverage in this second pathway, CMS officials said on a call with reporters.

Lee Fleisher, M.D., CMS chief medical officer and director of the Center for Clinical Standards and Quality, said the agency is particularly hoping that the broader reach of the analyses in the second pathway would allow for greater study into the effects off the drug class on a broad swath of patients.

Part of the agency's hesitancy in covering the class of drugs broadly is the lack of data on its clinical efficacy or potential harms in minority and underserved groups.

"We think it's critical that it's a diverse patient population," Fleisher said.

The national coverage determination will take effect immediately, and CMS could modify it in the future if clinical evidence dictates.

Aduhelm's approval in June 2021 was controversial within the healthcare industry, as many clinical experts expressed concern that data showing its efficacy was limited. Biogen, the drug's manufacturer, also set an eye-popping price tag of $56,000 per year for the drug, despite the concerns about whether it worked.

Biogen later halved the cost of Aduhelm amid sluggish sales.

Multiple providers and insurers have already vetoed the drug. While a number of private insurers stayed out of the fray, two of the industry's top lobbying groups, AHIP and the Blue Cross Blue Shield Association, previously applauded CMS' narrow approval proposal.

National coverage decisions like this are relatively rare, and Medicare determinations often serve as a bellwether for commercial insurers. CMS officials said they fielded more than 10,000 comments on the proposed coverage determination.