The Centers for Medicare and Medicaid Services announced Tuesday that it would cover Biogen’s controversial Alzheimer’s treatment that was approved by the Food and Drug Administration in June — but only for those who enroll in ongoing clinical trials.
The proposed rule means that many older Americans with early-stage Alzheimer‘s disease will not have access to the drug, which targets amyloid build-ups in the brain thought to cause the disease.
FDA approved the drug on an accelerated pathway, which requires a fourth clinical trial to demonstrate that the drug actually slows cognitive decline. The two Phase III clinical trials the agency used to approve the drug showed it worked to decrease amyloid, but it wasn't clear if it helped maintain cognition.
Aduhelm also has safety concerns. In Biogen’s last clinical trials, some participants experienced a swelling or bleeding of the brain as the drug removed amyloid. The FDA‘s decision to approve the drug, a monoclonal antibody that needs to be infused once a month, led to the resignations of three members of the agency’s neurological drug advisory committee.
Medicare doesn't consider drug costs when making a coverage determination, and it typically covers FDA-approved drugs. But Aduhelm’s price — $28,200 per year — as well as concerns over its effectiveness left many uncertain how CMS would handle this decision.
In restricting access, Lee Fleisher, the chief medical officer and director of the Center for Clinical Standards and Quality for CMS, acknowledged that the move was somewhat unusual but necessary because the government still has much to learn.
"It's important to recognize that the FDA approved the drug under an accelerated approval program, ultimately looking at the safety of the drug, with the approval based upon the surrogate outcome of reducing brain amyloid," he said during in a press conference. "This is unique, a more recent program. So, CMS is using this authority provided by Congress to determine if the drug is considered reasonable and necessary, meaning that the benefits of improvement of cognition outweigh the harms in the Medicare population."
CMS also proposes covering the cost of one amyloid PET scan per participant in these studies, which verifies that a person has the clumps of proteins Aduhelm targets. Medicare doesn't cover those scans at the moment, but said that it is still deliberating whether it should. Even with these costs covered, Biogen or another partner would be responsible for covering the rest of the costs of carrying out the trial.
The draft coverage determination for Aduhelm would apply to all monoclonal therapies for Alzheimer's disease that target amyloid. While Biogen’s drug is currently the only one to receive FDA approval, several other companies have similar drugs in late-stage clinical trials.
The decision was swiftly criticized by Biogen, which said it minimized the burden Alzheimer’s patients face.
"This will significantly limit patient access to an FDA-approved treatment, especially for underserved patients as evidenced in other [coverage with evidence development] determinations,” said a spokesperson for the drug company.
A comment period on the draft decision is now open for 30 days, and CMS will issue its final coverage determination decision by April 11.