Medicare Coverage of Aduhelm Will Be Limited to Patients in Trials

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by Marisa Wexler MS |

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The Centers for Medicare and Medicaid Services (CMS), in a final national policy decision, is allowing Medicare to cover the cost of Aduhelm (aducanumab) and other medicines in its class given accelerated approval only for Alzheimer’s disease patients enrolled in qualifying clinical trials.

Should Adulhelm or future amyloid-targeting monoclonal antibodies be given standard or full — rather than accelerated — FDA approval, CMS will broaden Medicare’s coverage to include people on those treatments enrolled in real-life studies “with comparators,” like large patient registry studies.

Standard approval for Adulhelm, however, could be years away.

“This final National Coverage Determination reflects CMS’ commitment to provide the American public with a transparent, trusted, evidence-based decision — without regard to cost — that is made only after a thorough analysis of public feedback,” Chiquita Brooks-LaSure, CMS administrator, said in a press release.

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“Through this decision, we are creating a pathway for people with Medicare to quickly access drugs the FDA determines have shown a clinical benefit and encourages manufacturers and trial administrators to ensure that the clinical trials recruit racially diverse participants,” Brooks-LaSure added.

The decision met with disappointment from the Alzheimer’s Association, which called the policy “broad overreach” by the CMS. It “essentially reject[s]” the FDA’s accelerated approval pathway for monoclonal antibody disease treatments, the group argued.

“CMS has created unnecessary barriers for individuals with Alzheimer’s disease. Patients with Alzheimer’s, a fatal disease, should have FDA-approved treatments covered by Medicare just as those facing other diseases do,” Harry Johns, the association’s CEO, said in a separate release.

Aduhelm, developed by Biogen and Eisai, is a monoclonal antibody that is designed to clear amyloid plaques — irregular clumps of protein in the brain that are characteristic of Alzheimer’s, and are thought to drive the disease. Clinical trial data have demonstrated that Aduhelm can lower amyloid levels, but the therapy’s effect on cognitive outcomes remains unclear, as two large Phase 3 trials found conflicting results.

In a controversial decision, the U.S. Food and Drug Administration (FDA) last year approved Aduhelm to treat early Alzheimer’s through its accelerated approval pathway. Essentially, this decision allowed Aduhelm to be sold in the U.S. based on the early data of amyloid clearance, while requiring the therapy’s developers to conduct additional studies to test its efficacy for cognition.

Biogen is planning to launch a Phase 4 clinical trial called ENVISION in the coming months to further test the therapy.

The CMS issued a draft National Coverage Determination (NCD) in January, stating that Medicare (the government-funded health insurance program for the elderly) would only pay for the cost of Aduhelm for people enrolled in clinical trials. This proposal was met with outrage by the Alzheimer’s Association and other patient organizations, and also drew harsh criticism from Biogen and health experts.

The final NCD decision stipulates that Medicare will cover the cost of Aduhelm and other amyloid-targeting monoclonal antibodies given accelerated approval only for people taking part in FDA- or National Institutes of Health (NIH)-approved clinical trials.

“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,” said Lee Fleisher, CMS chief medical officer and director of its Center for Clinical Standards and Quality.

“In arriving at this final decision, we looked at the very unique circumstances around this class of treatments and made a decision that weighed the potential for patient benefit against the significance of serious unknown factors that could lead to harm,” Fleisher added.

For amyloid-targeting antibodies given full FDA approval at a future date, Medicare will cover their cost for patients in a wider range of studies, including large registries collecting data on a therapy’s use in real-world settings.

“If a drug in this class shows evidence of clinical benefit through the traditional FDA approval process, then CMS will provide broad access and ensure the results from the rigorous trials are generalizable for people with Medicare participating in a CMS-approved study, such as a registry … We structured this decision to provide flexibility and assurance that CMS can respond quickly to providing coverage for any new drugs in this class when a clinical benefit is determined,” Fleisher said.

This decision, which differs from the original draft, was commended by the Alzheimer’s Association, which noted that similar registries have been implemented with success for heart disease and cancer treatments. The association, working with other institutes, also announced plans to open a large registry — the National Treatment and Diagnostic Alzheimer’s Registry — in November.

Still, the association maintains that the CMS, in its final decision, “has essentially ignored the needs” of Alzheimer’s patients and their families.

“People living with MCI [mild cognitive impairment], Alzheimer’s disease and other dementia deserve the same access to therapies given to those living with other conditions like cancer, heart disease and HIV/AIDS. They deserve the opportunity to assess if a FDA-approved treatment is right for them,” said Joanne Pike, the association’s  president. “Drugs that treat people in the early stages of Alzheimer’s could mean more time for individuals to actively participate in daily life, have sustained independence and hold on to memories longer.”

The association also expressed concerns that the CMS policy may make drug developers hesitant to invest in potential Alzheimer’s treatments that may not be covered even if they are approved by the FDA.

“The decision by CMS is a step backward for families facing Alzheimer’s disease,” said Maria Carrillo, PhD, chief science officer at the Alzheimer’s Association. “Years of increased research funding has led to more progress and innovation than ever before, but today’s decision may halt this progress as developers question if there is a pathway forward to coverage.”