The ongoing process to get Aduhelm, a new IV treatment for Alzheimer’s, approved and in use has been troubled and could continue to hit roadblocks.
Aduhelm – also known as aducanumab – is made and marketed by Massachusetts-based Biogen and is promoted as a first-of-its-kind treatment for the neurodegenerative disease. Trials showed the drug was effective at reducing plaque in the brains of Alzheimer’s patients, but further research must be done to determine its effectiveness in reducing the disease’s other symptoms.
Since the U.S. Food and Drug Administration approved Aduhelm this past summer, there has been a congressional investigation, an internal investigation and a related rise in Medicare Part B premiums. Questions about the effectiveness and high price tag of the drug remain, but Biogen has tried to address the latter by slashing the treatment’s cost in half.
FDA’s fast-track approval of Aduhelm raises concerns
The FDA used its accelerated approval process to approve Aduhelm to treat Alzheimer’s on June 7, 2021, citing its ability to reduce amyloid beta plaque. The exact cause of Alzheimer’s is unknown, but buildup of that protein plaque in the brain is an indicator of the disease that affects almost six million Americans, according to the Centers for Disease Control. Even though the treatment is approved, the accelerated approval requires more research to be done on its effectiveness. Biogen says these studies are set to begin in the spring of 2022.
Andrew Duxbury, MD, a geriatrician at the University of Alabama Birmingham, said the FDA can withdraw its approval of Aduhelm if Biogen cannot prove it improves memory or mitigates other Alzheimer’s symptoms.
“They were not able to produce for the FDA any evidence that the drug actually has any clinical effect,” Duxbury said. “Before I would be prepared to ask patients to undergo potential issues and expense of the kind that are being talked about with this drug, I want to see some better data that it’s going to do some good.”
NPR reports the FDA went against the advice of its own advisory board, which recommended against the approval, and the process resulted in three members resigning from the board. Less than three weeks after the approval, two U.S. representatives announced a Congressional committee investigation into the drug and the process. The following month, in July 2021, the acting head of the FDA, Janet Woodcock, MD, publicly requested an investigation into the matter by the Inspector General of the U.S. Department of Health and Human Services. The IG’s office obliged, but that report is not expected until 2023.
Side effects and cost raise concerns
As far as the side effects of the drug, Duxbury singled out brain bleeding as one of the issues Aduhelm can cause. He said patients must undergo MRI scans to ensure no brain bleeding is happening during treatment.
The price of the treatment is also high: A year’s worth was originally estimated at about $56,000, but Biogen cut the drug’s price by about half in December. A maintenance dose for a patient of average weight — 163 pounds — will run $28,200 annually. This cut came after Biogen reported only $300,000 in sales for the third quarter of 2021, the first full quarter the drug had its approval.
Medicare Part B and the novelty of Aduhelm
Aduhelm is the first new drug approved by the FDA for the treatment of Alzheimer’s since 2003, and Duxbury says it differs from every other drug available.
“It is actually attacking the actual pathology of the disease,” he said. “None of the other current medical treatments we’ve got do that.”
The new, expensive treatment will have an effect on Medicare, which the AP reports is expected to be one of the primary purchasers of the drug. In November, when the Centers for Medicare and Medicaid Services announced a historic increase of Medicare Part B monthly premiums for 2022, the organization specifically cited Aduhelm – and the uncertainty around it – as a reason for the increase. The $170.10 premium for 2022 is an increase of more than $20 over 2021, the biggest year-over-year hike in nominal dollars in Medicare’s history.The number of patients who could benefit from Aduhelm may be limited as well. After its approval, the FDA clarified the drug is best-suited for patients with mild or early-stage Alzheimer’s. Despite its issues in the United States (as well as a recent refusal in Japan), a second country, the United Arab Emirates, approved the use of Aduhelm in October, according to Dubhai’s Khaleej Times.