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Our Take: Final CMS policy: Aduhelm will be covered only in clinical trials

Apr 18, 2022

As CMS proposed in January, the agency’s final national coverage policy covers Aduhelm (aducanumab), Biogen’s drug for Alzheimer’s disease, only for Medicare beneficiaries who are participating in qualifying clinical trials.

Specifically, the national coverage determination (NCD) covers FDA-approved monoclonal antibodies that target amyloid for the treatment of Alzheimer’s disease, when furnished in accordance with certain coverage criteria, for patients with a clinical diagnosis of mild cognitive impairment due to Alzheimer’s disease or mild dementia, both with the confirmed presence of amyloid beta pathology.

The NCD distinguishes between drugs approved through the accelerated process, as Aduhelm was (i.e., using a surrogate endpoint, such as amyloid reduction, to demonstrate that there is a reasonable assurance of clinical benefit), and those that might be approved in the future through the traditional process (i.e., based on evidence of efficacy from a direct measure of clinical benefit).

Coverage for drugs that receive accelerated approval will be limited to patients enrolled in clinical trials approved by the FDA or the National Institutes of Health. Regarding these trials, the final NCD is not as restrictive as the proposed version was. For instance, it allows for the trials to be conducted in settings other than hospitals. CMS also eased the exclusions on certain patient populations, such as individuals with Down syndrome.

Under the final NCD, drugs approved through the traditional process may be eligible for broader coverage. This change from the proposed NCD is intended to allow for flexibility in “a less rigorous study design” — including data generated through routine clinical practice or a registry.

If Biogen’s ongoing confirmatory trial provides evidence that Aduhelm can slow the progression of Alzheimer’s disease, then Aduhelm will be eligible for broader coverage as well.

After CMS released the proposed NCD earlier this year, the agency accepted public comments for 30 days. In making its final decision, CMS reviewed more than 10,000 stakeholder comments and over 250 peer-reviewed documents.

CMS Administrator Chiquita Brooks-LaSure told The New York Times that the agency’s decision to limit coverage for Aduhelm is intended to protect patients while more data can be gathered to show whether Aduhelm can truly slow the progression of cognitive decline.

She also said the “vast majority” of the comments CMS received during the public comment period were in favor of limiting coverage of the drug “to a really controlled space where [CMS] could continue to evaluate its appropriateness for the Medicare population.”

Our Take: Rarely has a drug approval led to the level of controversy surrounding the FDA’s approval of Aduhelm.

In granting the accelerated approval, the agency went against the recommendation of its advisory panel. Pointing to a lack of evidence that Aduhelm can effectively treat Alzheimer’s disease, along with concerns that some patients could experience harmful side effects, the panel chose not to recommend approval.

Experts on the panel also expressed concern that the FDA’s approval of Aduhelm might set a precedent, leading to the approval of other drug candidates that demonstrate only modest benefits.

Three panel members resigned soon after the drug was approved.

Biogen added considerable fuel to the fire when it priced Aduhelm at $56,000 for a year of treatment. The Institute for Clinical and Economic Review (ICER) said a fair price would be between $11,000 and $23,100. Following months of lackluster sales, Biogen announced in December that it was cutting the drug’s price in half, to $28,200.

Meanwhile, within weeks of Aduhelm’s approval, two House committees announced an investigation into both the approval of the drug and the drug’s price. Dr. Janet Woodcock, the acting commissioner of the FDA at the time, asked the Office of the Inspector General to conduct its own investigation.

Around that same time, to address substantial criticism, the FDA revised Aduhelm’s label to narrow the patient population. Whereas the original approval was for all patients with Alzheimer’s disease, the revised label recommended use of the drug only in patients with Alzheimer’s disease who have mild cognitive impairment or mild dementia.

Commercial insurers were reluctant to cover Aduhelm, with some saying they were going to wait and see what CMS decided to do before making any decisions of their own. Others said they would only cover the drug if prior authorization was obtained.

Hospitals were disinclined to administer Aduhelm infusions. Some wanted to wait until coverage decisions had been made. Some wanted to wait until the investigations had been completed or additional clinical evidence of the drug’s efficacy was available.

For patients and their families, the swell of controversy that followed Aduhelm’s approval had to be beyond frustrating. Aduhelm was the first new drug to be approved as a treatment for Alzheimer’s disease in nearly 30 years, but the price and lack of coverage held it out of reach for most patients.

After CMS first suggested that it would only cover Aduhelm for patients in clinical trials, patient advocacy organizations and lobbyists began pressuring the agency to change its stance. Various lawmakers on both sides of the aisle asked CMS to reconsider the coverage limitations, including a group of more than 40 members of Congress who wrote to the Department of Health and Human Services and CMS earlier this month.

But in the end, it looks like CMS has tried to correct what was largely seen as a mistake in approving Aduhelm when it did, by restricting Medicare coverage of the drug until there’s clear evidence that it actually works.

Clearly, there’s still more to be settled, including the ongoing investigations into the drug’s approval. In the weeks ahead, we’ll see how payers and providers respond to CMS’ final decision.

We’ll also see if CMS is going to reduce Medicare premiums, after increasing them sharply in anticipation of the hefty cost of covering Aduhelm for a larger swath of beneficiaries. The agency has said it’s considering a reduction, so we think it’s just a matter of time.

What else you need to know
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