The Food and Drug Administration (FDA) on Thursday approved an update to the prescribing label for a controversial new Alzheimer's drug in an attempt to narrow its intended use.
The agency recommended that Biogen's Aduhelm drug should only be used in patients with mild Alzheimer's dementia or mild cognitive impairment, a departure from the initial approval that was much broader.
The change, which was announced by Biogen, emphasizes that the drug was only studied in people with mild forms of the disease. It makes clear "there are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied."
However, the FDA's initial approval last month did not make any distinctions, and the agency at the time said the drug was meant to treat all forms of the disease.
The narrow label could make it cheaper for Medicare to cover the drug, which was priced at a staggering $56,000 a year per patient.
Physicians and drug pricing advocates criticized the high cost and broad approval, saying it could wreak havoc on Medicare spending, especially for a drug that had an unproven benefit. Since Alzheimer's disease primarily affects seniors who are eligible for Medicare, taxpayers will largely foot the bill for the new drug.
Under the narrower label, the company has estimated about 1.5 million people with mild Alzheimer's would be eligible for the drug.
Total Medicare spending for all prescription drugs was $37 billion in 2019, but that amount could be blown out of the water if Medicare paid for all eligible patients.
A Kaiser Family Foundation estimate showed if even 500,000 Alzheimer's patients on Medicare are prescribed Aduhelm, it would cost the program nearly $29 billion a year.
Alfred Sandrock Jr., head of research and development at Biogen, in a statement said the company submitted the labeling change to clarify the eligible patient population "based on our ongoing conversations with prescribing physicians, FDA and patient advocates."
But the decision to narrow the label was started at FDA. Agency officials approached Biogen and initiated meetings with the company to clarify labeling.
It's unusual for a company to agree to narrow a label for a new drug so soon after its approval, and critics said the move by FDA is an attempt to clean up a mess of its own making.
Aduhelm was the first new Alzheimer's drug approved in nearly 20 years, but the approval came over the near-unanimous objections of the FDA's expert advisory committee. Three of the agency's outside advisers resigned over the decision.
Aduhelm is not a cure for Alzheimer's, and the FDA made its decision without clear evidence that the drug can even slow the progression of the disease.
"A key question has been whether the Aduhelm prescribing information supports the treatment of patients at all stages of Alzheimer’s disease. Hearing these concerns, FDA determined that clarifications could be made to the prescribing information to address this confusion," the agency said in an e-mailed statement to The Hill.
Yet the FDA also appeared to leave the door open to broader use of the drug, despite saying its use should be limited.
"Some patients may benefit from ongoing treatment even if they progress from mild cognitive impairment or mild dementia to later stages of the disease during treatment with aducanumab," FDA said. "Prescribers should evaluate their individual patient’s response to treatment with aducanumab and determine if there is potential for benefit with continued treatment."
The treatment, which is delivered intravenously, is intended to reduce the amount of plaque in the brains of Alzheimer's patients. Brain plaques are thought to be a contributor to Alzheimer's disease.
But the FDA has acknowledged the drug did not demonstrate a clear clinical benefit in terms of actually slowing the progression of Alzheimer's. Instead, officials have argued that by removing the plaques, Aduhelm “is reasonably likely" to benefit patients, and could potentially slow cognitive decline.
The agency decided to approve the drug under its accelerated approval pathway, which is typically used for rare diseases or drugs with much smaller patient populations.
Two congressional committees in the House have launched an investigation into the FDA’s review of the drug. And lawmakers in the Senate have called for hearings into the drug’s cost and impact on federal spending.