Congressional report: FDA broke own protocols in approving Aduhelm 

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A staff report by two House committees points to alleged irregularities in the Food and Drug Administration's approval of the Alzheimer's drug Aduhelm.

"The High Price of Aduhelm's Approval: An Investigation into FDA's Atypical Review Process and Biogen's Aggressive Launch Plans" was released by the Committee on Oversight and Reform and Committee on Energy and Commerce after an 18-month investigation.

The FDA and drugmaker Biogen inappropriately worked together for approval of the Alzheimer's disease drug, aducanumab, known by its trade name, Aduhelm, according to a press release from Oversight and Reform Committee Chairwoman Carolyn B. Maloney, D-N.Y., and Energy and Commerce Committee Chairman Frank Pallone Jr., D-N.J. The press release can no longer be found online.

The FDA put the drug through an accelerated approval process, the release said. The FDA's interactions with Biogen were atypical and failed to follow the agency's own documentation protocol, it said. 

Biogen also initially set an "unjustifiably high price" for Aduhelm, at $56,000 per year, they said, to "make history" and "establish Aduhelm as one of the top pharmaceutical launches of all time," according to the report.

"Documents obtained by the committees show that Biogen viewed Aduhelm as an unprecedented financial opportunity – estimating a potential peak revenue of $18 billion per year – and developed aggressive launch and marketing plans to maximize revenue throughout the drug's lifecycle," the release said.

Effective January 1, 2022, Biogen cut the wholesale acquisition cost of Aduhelm by 50%, from an estimated $56,000 a year to $28,200 a year. 

WHY THIS MATTERS 

Neither the committees' press release nor report appears to give a reason as to why the FDA allegedly wanted to fast-track approval of Aduhlem.

Biogen said in a statement that it stands by the integrity of the actions it has taken.

"As stated in the congressional report, an FDA review concluded that, 'There is no evidence that these interactions with the sponsor in advance of filing were anything but appropriate in this situation,'" Biogen said.

Medical professionals and the federal government have scrutinized Aduhelm as to its efficacy. In July 2021, the American Journal of Managed Care questioned whether the decision for FDA approval in June 2021 was ethical.

Last year, the Centers for Medicare and Medicaid Services limited Medicare beneficiaries' access to Aduhelm to those enrolled in qualifying clinical trials citing its questionable clinical benefit. AHIP, the association for insurers, agreed that more clinical data was needed.

But UsAgainstAlzheimer's said that limiting the use of Aduhelm, the only FDA-approved monoclonal antibody treatment to target amyloid in the brain for the treatment of Alzheimer's disease, was unacceptable. 

Others such as the Campaign for Sustainable Rx Pricing questioned its price tag.

Biogen expected Aduhelm to be a burden to Medicare and costly to patients, according to the House committees' report.

Aduhelm's pricing was based on strategic guidance from third-party consultants who suggested a price greater than $40,000 per year would maximize revenue, while a price less than $40,000 per year would limit both payer and physician pushback on the price, the release said.

"Internal company documents show that Biogen was aware the financial burden of its high price for Aduhelm would fall primarily on Medicare," the release said. "Biogen estimated that the drug would cost Medicare $12 billion in one year – representing 36% of Medicare's 2018 Part B budget. Documents also show that Biogen knew from early pricing models that some Medicare patients would struggle to afford Aduhelm." 

THE REPORT

FDA staff and Biogen engaged in at least 115 meetings, calls and substantive email exchanges over a 12-month period beginning in July 2019, according to the report. 

Despite FDA guidance, there was no clear record of the informal meetings and other interactions between agency staff and Biogen. 

The report found that FDA and Biogen staff inappropriately collaborated on a joint briefing document ahead of a November 6, 2020, Peripheral and Central Nervous System Drugs Advisory Committee meeting. This afforded Biogen advance insight into FDA's responses.

The FDA granted approval for the drug under an accelerated approval pathway and accepted a broad label indication for Aduhelm, despite lack of clinical data on disease stages other than mild cognitive impairment and mild Alzheimer's disease. 

Internal documents show that Biogen accepted this broad indication statement for Aduhelm, despite internal reservations about the lack of evidence of clinical benefit for patients at disease stages outside of the clinical trials.

THE LARGER TREND

More than six million people in the United States live with Alzheimer's disease, a number projected to increase to as many as 14 million people by 2060, according to the House committees' press release.

The FDA granted accelerated approval for Aduhelm on the basis that the drug reduces amyloid beta plaque in the brain. The FDA's action came despite the fact that Biogen canceled clinical trials for Aduhelm in March 2019 due to an independent report indicating the drug was unlikely to effectively slow cognitive and functional impairment and that further clinical study would be futile, the release said.

"Despite the advisory committee's lack of recommendation for approval, as well as internal concerns raised by experts about the inconsistency of the drug's clinical data, FDA granted accelerated approval to Aduhelm on June 7, 2021, based on Aduhelm's effect on a proxy for clinical benefit. Following the drug's approval, several members of the FDA advisory committee publicly resigned in protest," the release said.

Twitter: @SusanJMorse
Email the writer: SMorse@himss.org