The US Centers for Medicaid and Medicare Services (CMS) released a national policy for coverage of Biogen’s/Eisai’s Aduhelm (aducanumab) and any future monoclonal antibodies approved by the US Food and Drug Administration as anti-beta-amyloid drugs for treating Alzheimer’s disease. The CMS had issued a draft decision earlier this year (2022) and has now issued a final decision in a two-part National Coverage Determination.

Under the first part of its decision, the CMS says that Medicare, the US federal health insurance program for people over age 65, will cover monoclonal antibodies that target amyloid (or plaque) for treating Alzheimer’s disease that receive traditional approval from the FDA under coverage with evidence development. CMS will provide enhanced access and coverage for people with Medicare participating in CMS-approved studies, such as a data collection through routine clinical practice or registries. Registry data may be used to assess whether outcomes seen in carefully controlled clinical trials (e.g., FDA trials) are reproduced in the real-world and in a broader range of patients.  Any new drugs in this class that receive FDA traditional approval may be available in additional care settings that people with Medicare can use, such as an outpatient department or an infusion center.

In the second part of its decision, for drugs that FDA has not determined to have shown a clinical benefit or that receive an accelerated FDA approval, Medicare will cover participants only in approved clinical trials by the FDA or National Institutes of Health. FDA’s accelerated approval pathway provides patients with a serious disease earlier access to drugs when there is an expectation of clinical benefit despite some uncertainty about the clinical benefit. The accelerated approval pathway requires companies to verify clinical benefit in a post-approval trial. If the sponsor cannot verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug. For people with Medicare who are participating in these confirmatory trials, CMS will cover the drug and any related services. The decision is specific to individuals who have a clinical diagnosis of mild cognitive impairment due to Alzheimer’s disease or mild dementia with a confirmed presence of plaque on the brain.

Biogen’s/Eisai’s Aduhelm was approved under FDA’s accelerated approval pathway in June 2021, and based on the CMS decision, Medicare coverage for the drug will be limited to those participants in the confirmatory trials.

“This unprecedented CMS decision effectively denies all Medicare beneficiaries access to Aduhelm (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs,” said Biogen in an April 7, 2022, statement. “It may also limit coverage for any future approved treatment in the class. These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas. When additional data from this new class of treatments become available, Biogen urges CMS to reconsider today’s [April 7, 2021] decision for all FDA-approved amyloid-beta targeting therapies.”

Biogen says it is considering its options and says it will provide updates as it further evaluates the business impact of the CMS decision. Biogen submitted the study protocol for the confirmatory trial for Aduhelm to the FDA in March 2022. Biogen expects the first patient to enter screening in May 2022, and the trial’s primary completion approximately four years after the study begins. The study will be a global, placebo-controlled trial, aiming to enroll around 1,500 patients with early Alzheimer’s disease and confirmation of amyloid beta pathology.

Biogen’s/Eisai’s Aduhelm was slated for blockbuster status by some analysts, but thus far the drug has performed below expectations. Following accelerated approval in June 2021, Biogen posted full-year 2021 sales of $3 million.

From 2017 to March 13, 2022, Biogen and Eisai jointly collaborated on the development, commercialization and manufacturing of Aduhelm. Effective March 14, 2022, Biogen has sole decision-making authority over the development, commercialization, and manufacturing of the drug. In 2022 the parties will continue in a global profit/loss sharing arrangement subject to a cap on Eisai’s expenses for 2022. Eisai will be entitled to a tiered royalty on net sales of the drug as of January 1, 2023.

Source: Centers for Medicare & Medicaid Services and Biogen