Probe: Alzheimer’s drug approval flawed
The biotechnology company Biogen and its regulator, the Food and Drug Administration, worked in concert, ignoring internal concerns from the company and skirting the agency’s own written guidance, to allow the Alzheimer’s treatment Aduhelm to receive accelerated approval and hit the market at a cost to patients of $56,000 a year, according to a scathing report released Thursday by two House committees.
The “unusual” collaboration, which resurrected Aduhelm three months after Biogen had canceled clinical trials, unfolded through at least 115 meetings, calls and email exchanges between the company and the FDA in a year, said the report by the Committees on Oversight and Reform, and Energy and Commerce.
The joint effort climaxed with staff from the agency helping Biogen draft a document used to brief the FDA’s own advisory committee before it met to discuss Aduhelm on Nov. 6, 2020. Although the FDA often follows an advisory committee recommendation, it did not this time. After no member of the advisory committee recommended Aduhelm, the FDA changed course, allowing Biogen to move its drug to an accelerated approval process.
At the FDA’s suggestion, the drug was labeled for use by the nation’s more than 6 million Alzheimer’s patients, even though it had been tested only on people with early Alzheimer’s and
mild symptoms, the report said.
“It’s the worst decision the FDA has ever made” in the last half-century, said Sidney Wolfe, founder of the advocacy group Public Citizen’s Health Research Group. “It was an unprecedented alliance between the company and the FDA.”
“We fully cooperated with the Committees’ evaluation and we continue to review their findings and recommendations,” the FDA said in a statement responding to the report. “It is the agency’s job to frequently
interact with companies in order to ensure that we have adequate information to inform our regulatory decision-making. We will continue to do so, as it is in the best interest of patients. That said, the agency has already started implementing changes consistent with the Committee’s recommendations.”
The agency had previously conducted an internal investigation of its handling of Aduhelm, concluding more than a year ago that although the collaboration “exceeded the norm in some
respects,” there was “no evidence” that dealings between the company and regulator “were anything but appropriate.”
The internal report said the decision to work “proactively” with Biogen “is consistent with FDA policy” in light of both “the large unmet medical need” for Alzheimer’s treatment, and an FDA official’s view that one of the Aduhelm studies could represent “a home run” as far as safety and effectiveness.
The report by the two House committees faulted the company too, saying that Biogen knew the initial $56,000 per year price ― reduced to $28,000 in January 2022 ― would put a heavy burden on patients. But the Cambridge, Mass.- based company estimated the treatment could earn them as much as $18 billion per year and exulted in a slide presentation to its board: “Our ambition is to make history ... to establish (the drug) as one of the top pharmaceutical launches of all time.”
In a statement responding to the report, Biogen said it had cooperated with the committees and “stands by the integrity of the actions we have taken.” Biogen’s statement also cited the FDA’s internal investigation, which concluded that there was no evidence of impropriety in the dealings between the agency and company.
Biogen stuck with the $56,000 per year price tag despite projections that the drug could wind up costing Medicare up to $12 billion in a single year. Other Alzheimer’s sell for much less. A year’s supply of Aricept costs less than $ 8,000; Exelon, a drug in the same family, costs about $8,800 a year’s supply; and Namenda costs less than $3,000 per year.
The committees recommended that the FDA document all its communications with drug sponsors, establish a system for partnering with companies to produce the reports used to brief its own advisory committees and update its formal guidance for developing and reviewing new Alzheimer’s drugs.