Final CMS decision limits coverage of Biogen’s Aduhelm to those in clinical trials

Final CMS decision limits coverage of Biogen’s Aduhelm to those in clinical trials

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Medicare will only cover Biogen Alzheimer’s disease drug Aduhelm for beneficiaries enrolled in a clinical trial, a decision that limits use of that product and also affects experimental medications still in development for the neurodegenerative disorder. That’s because this coverage determination extends to antibody drugs in the same class, and agency coverage decisions are also guideposts for payers as they weigh whether to reimburse the Biogen drug and additional Alzheimer’s medicines that may follow.

In explaining the final coverage determination announced late Thursday, officials at the Centers for Medicare and Medicaid Services said the decision reflects the available data for Aduhelm as well as the need to find additional evidence about the drug’s benefit and risks.

“There is the potential for promise with this treatment; however, there is not currently enough evidence of 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,” Lee Fleisher, CMS chief medical officer and director of the Center for Clinical Standards and Quality, said in the agency’s announcement.

The final CMS decision for Aduhelm ends a coverage determination process that began shortly after the FDA granted accelerated approval for the Biogen drug last June. Accelerated approval is a pathway intended to more quickly bring to the market drugs that address major unmet medical needs. This pathway requires less evidence than a standard drug review. Accelerated approval decisions are based on a surrogate endpoint, which is an indication that a drug might be working even if patient benefit isn’t clear.

For Aduhelm, that surrogate endpoint is the reduction in amyloid beta. Aduhelm is an antibody drug designed to break up amyloid plaques. While the buildup of amyloid is characteristic of Alzheimer’s, it’s still unclear whether reducing these plaques helps patients, and Biogen’s own clinical data offer a mixed picture. An independent analysis of interim Phase 3 data in 2019 concluded the clinical trials were unlikely to succeed. But Biogen conducted a new analysis that it said showed success in one of the two pivotal studies. On that basis, the company sought FDA approval.

The final CMS determination covers Aduhelm and other amyloid-targeting antibody drugs under what’s called coverage with evidence development. This CMS provision allows Medicare to cover a drug in a clinical trial designed to generate additional data that could support broader coverage. Coverage with evidence development has been applied previously; examples include stem cell transplants for blood disorders and some medical devices, such as cochlear implants.

The CMS decision largely tracks with the proposal the agency issued in January. At that time, the agency said it would cover Aduhelm only for patients in a CMS-approved, randomized, and controlled clinical trial. The final decision broadens that trial requirement a bit to include comparative studies that have a range of study designs. These data may be collected through routine clinical practice, also known as a registry.

The CMS decision removes some requirements that the agency initially proposed in January. For example, criteria that would have excluded some groups of patients, such as those with Down’s syndrome, are no longer in the final coverage determination. CMS said it removed the exclusion criteria based on public comment. The agency also removed a provision requiring the approved clinical trials to take place in hospital-based outpatient facilities. That proposed requirement was based on safety concerns. Severe side effects of Aduhelm include swelling and bleeding in the brain. CMS said the removal of this requirement was also based on public comment, and will enable clinical research to provide data about patient outcomes from a broader range of community settings.

The decision to restrict coverage of Aduhelm to those in clinical trials is the latest in a string of setbacks for the drug and Cambridge, Massachusetts-based Biogen. Market uptake of Aduhelm, initially priced at $56,000 a year, has been slow. Despite the prevalence of Alzheimer’s and the patient need for new treatments, the drug fell well short of blockbuster expectations, generating just $3 million in 2021 sales, according to Biogen’s financial statements. Biogen slashed the drug’s price in half starting Jan. 1, a decision the company said would make the drug more accessible to more people.

In a statement issued after the CMS announcement, Biogen said the coverage determination effectively denies all Medicare beneficiaries access to Aduhelm. The company urged the agency to reconsider the decision after additional clinical data from this class of drugs become available. Eisai, Biogen’s partner in developing Alzheimer’s drugs, is following the accelerated approval pathway used for Aduhelm for a different antibody drug, lecanemab. Phase 3 data for that drug are expected this fall. In its own statement, Eisai said it believes the design for this study could meet the evidence criteria set by the CMS in its coverage determination. The company added that if the data are positive, the results create the potential for CMS to reconsider full coverage of lecanemab if that drug secures full FDA approval.

CMS pushed back against suggestions that its coverage decision blocks the research and development of innovative Alzheimer’s therapies. The agency said its decision facilitates innovation by providing clarity on the criteria for Medicare coverage. Under the law, CMS must cover medical products and services deemed “reasonable and necessary.” CMS said that its final decision outlines what evidence Alzheimer’s medications in this drug class need to show to meet this standard.

“Medical innovation must include evidence that demonstrate health outcomes to patients in consideration with potential harms,” the agency said in a fact sheet with additional detail about the decision.

Photo: Scott Eisen/Bloomberg, via Getty Images

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