- CMS mostly finalized previously proposed criteria for covering Aduhelm, which has been promoted as the first drug to treat the underlying pathology of Alzheimer’s disease.
- The coverage criteria have been expanded to include additional types of clinical trials and settings.
- State Medicaid programs are statutorily obligated to cover the drug but can apply utilization-management processes.
Aduhelm didn’t pass muster as a product that should be widely covered by Medicare, but the new Alzheimer’s disease drug and future drugs in the same class still can prove themselves worthy of comprehensive coverage.
That was the primary takeaway from CMS’s recent coverage announcement and comments by agency leaders, who emphasized that they sought to make the decision-making process transparent and evidence-based.
The final coverage determination in large part adheres to the proposed criteria that were announced in January, after which more than 10,000 comments were submitted by stakeholders. Tweaks to the proposed coverage determination are designed to modestly expand coverage of the drug while its efficacy is studied.
The decision prevents coverage of Aduhelm in most healthcare encounters in response to the widespread concerns about the drug’s safety and effectiveness that arose leading up to the FDA approval's on an accelerated basis last June.
One expansion of the proposed criteria applies to Aduhelm (the brand name for the drug aducanumab) and any other monoclonal antibody treatments that receive accelerated approval by the FDA and are designed to reduce amyloid in the brain. Several such treatments are in development.
Medicare will cover the administration of all such drugs in randomized controlled trials authorized by the FDA or National Institutes of Health (NIH), deviating from the proposal to require that all trials be approved by CMS.
“CMS will gather additional evidence on the effectiveness and safety of this drug by supporting rigorous studies by the FDA and NIH to help determine if there truly is a clinical benefit for Medicare beneficiaries,” Lee Fleisher, MD, chief medical officer and director of CMS’s Center for Clinical Standards and Quality, said during a stakeholder call. “For drugs in this class, future drugs that are in the pipeline and demonstrate clinical benefits will be covered so that patients have access.”
Treatments that receive full FDA approval can be covered in CMS-approved studies, including those involving data collection through routine clinical practice or registries. That’s an expansion of the proposed criteria, which would have restricted coverage to randomized clinical trials.
“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,” CMS stated in a news release.
CMS also removed proposed restrictions on clinical-trial coverage that would have excluded patients with neurological conditions other than Alzheimer’s disease and would have required all approved trials to be conducted in hospital-based outpatient settings.
“We intend to be nimble and flexible as new evidence is developed and comes out,” said Tamara Syrek Jensen, JD, director of the coverage and analysis group in the Office of Clinical Standards and Quality. “We can always reconsider the coverage determination.”
A much-anticipated decision
The decision-making process attracted attention because of the high-profile nature of the drug, which was touted as the first treatment that addresses the underlying pathology of Alzheimer’s disease. The manufacturer, Biogen, didn’t endear the product to policymakers and stakeholders when it initially set a price of $56,000 per treatment after the FDA approved the drug in June. Six months later, Biogen cut the price in half in response to pushback.
Among those who spoke out about Aduhelm following FDA approval, a preponderance of providers and insurers supported coverage restrictions. For example, Cleveland Clinic, Mount Sinai and Providence quickly said they wouldn’t administer the drug until more clinical evidence was available. According to a Bloomberg report, 25 large insurers said they didn’t deem Aduhelm to be medically necessary, although Humana said it planned to cover the drug for members who resemble those whom the treatment benefited in clinical trials.
On the other side, advocacy groups for patients with Alzheimer’s disease spent millions of dollars on campaigns urging Medicare to expand coverage, according to a Reuters report. After CMS announced its final decision, the Alzheimer’s Association expressed disappointment, stating that the agency “has essentially ignored the needs of people living with Alzheimer’s disease.”
“While we note CMS has expanded where those clinical trials may take place, in reality this remains an unnecessary and never-before-imposed barrier to access an FDA-approved treatment,” the association added.
The need for a risk-benefit analysis
The drug is targeted to patients who have mild cognitive impairment and may not have outward signs of dementia. CMS leaders said that because those patients can still lead healthy and active lives, a rigorous evaluation of the risks and benefits is all the more important.
“As a physician, I know there may be potential for promise with this treatment,” Fleisher said. “However, while the drug is known to reduce amyloid plaque in the brain, there is not currently evidence that this reduction leads to a clinical benefit in the form of improved cognition or function.”
Meanwhile, he added, known risks include potentially fatal brain bleeds.
Considering the various factors, “There is not currently sufficient evidence to support full coverage of this drug for people with Medicare until the statutory reasonable and necessary standard is met,” he said.
A different story in Medicaid
Because Biogen is in the Medicaid Drug Rebate Program and Aduhelm has been approved by the FDA, state Medicaid programs must cover the drug broadly and can’t limit coverage to clinical trials, said John Coster, director of the Division of Pharmacy in CMS’s Center for Medicaid and CHIP Services.
However, he said, states can “subject the drug to traditional utilization-management techniques, such as prior authorization and step therapy.”
In addition, coverage is not required for beneficiaries who are dually eligible for Medicare and Medicaid.