April 8, 2022 -- Federal officials have made their final decision: Medicare will only pay for patients to get the new Alzheimer's drug aducanumab (Aduhelm) if the patients are participating in clinical trials.
On Thursday, the Centers for Medicare & Medicaid Services (CMS) released its national coverage determination for aducanumab and other similar drugs that target beta-amyloid in the brain, which is considered a hallmark of Alzheimer's disease.
In a press release, the CMS said its decision is a two-part policy based on the currently available evidence for anti-amyloid agents.
Last year, the FDA approved aducanumab for Alzheimer’s disease using the accelerated approval pathway, because trials had shown the drug removed beta-amyloid from the brain, but it wasn’t clear the drug had improved the symptoms of people with Alzheimer’s.
The decision sparked controversy and led to the resignation of three members of the FDA's advisory panel that had voted against approving the drug.
CMS set far less restrictive conditions for potential coverage of anti-amyloid drugs that may get full FDA approval in the future. In such cases, coverage would be pegged to participation in CMS-approved studies, such as a data collection through routine clinical practice or registries, not only clinical trials.
Howard Fillit, MD, co-founder and Chief Science Officer of the Alzheimer’s Drug Discovery Foundation, supported the decision.
"Medicare will now provide coverage for patients enrolled in any FDA- or NIH-approved trial,” Fillit said. “This is an important change that will expand access for more patients to access the drug in additional settings."
"Although this is an improvement, it would have been preferable for CMS to include patient registries, which collect real-world evidence that more closely reflects the actual population living with Alzheimer's, compared to the limited populations that enroll in clinical trials,” he added. “This approach would also have extended coverage of Aduhelm to more patients currently living with Alzheimer's disease."
He also welcomed the flexibility CMS outlined for future coverage of drugs with more compelling data.
"Drugs in the same class are not necessarily equal and each should be evaluated and made available to patients on its own merits," Fillit said.
In a statement, aducanumab manufacturer Biogen disagreed with CMS' limit on coverage for the drug and protested the two-tiered approach created for amyloid-targeting antibody drugs.
"These coverage restrictions, including the distinction between accelerated approval and traditional approval, have never been applied to FDA-approved medicines for other disease areas," Biogen said.
Biogen and other critics of the CMS decision contend it denies patients access to a medication that might slow Alzheimer’s-related mental decline, while supporters question whether showing the drug removed beta-amyloid from the brain means it helps patients’ symptoms.
CMS officials said the agency tried to make the process leading to today's decision transparent and evidence-based. It noted it gathered more than 10,000 stakeholder comments on its draft decision and considered more than 250 peer-reviewed documents.
"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," CMS Administrator Chiquita Brooks-LaSure said in a statement.
"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," she added.