FDA’s Approval Process of Controversial Alzheimer’s Drug Was ‘Rife With Irregularities’: Congressional Probe

A vial and packaging for the drug Aduhelm is shown on June 7, 2021. Biogen via AP

The U.S. Food and Drug Administration’s (FDA) approval process of the controversial and costly new Alzheimer’s drug Aduhelm was “rife with irregularities” and “highly atypical,” according to a report released on Dec. 29 by the House Oversight and Energy and Commerce committees.

The report (pdf) is the result of an 18-month investigation conducted by the Committee on Oversight and Reform, and Committee on Energy and Commerce into the regulatory review and approval, pricing, and marketing of the Massachusetts-headquartered biotechnology company Biogen Inc.’s Alzheimer’s disease drug, aducanumab, which is widely known by the name Aduhelm.

Aduhelm was granted accelerated approval by the FDA in June 2021 after data from clinical trials showed a reduction in the amyloid-beta plaques of the brain, which “is expected to lead to a reduction in the clinical decline of this devastating form of dementia.”

Experts believe brain plaques could be a contributor to Alzheimer’s.

However, the report published on Thursday noted that the approval 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.”

An outside advisory committee of experts also recommended against approving the drug, the report noted.

An FDA advisory committee strongly recommended against Aduhelm’s approval because the drug didn't test well. Daisy Daisy/Shutterstock

Medicare Restricts Coverage of Aduhelm

The Medicare program has restricted coverage of Aduhelm, proposing instead that it only be used in clinical trials, which has led to severely limited use of the Biogen drug.

Specifically, the report states that the FDA’s Interactions with Biogen were “atypical” and that the agency failed to follow its own protocol.

“Documents obtained by the Committees show that FDA staff and Biogen engaged in at least 115 meetings, calls, and substantive email exchanges over a 12-month period beginning in July 2019. These exchanges included at least 40 FDA-Biogen ‘working group’ meetings. FDA’s own internal review of the agency’s approval process for Aduhelm found that the extent of collaboration between FDA and Biogen was atypical and ‘exceeded the norm in some respects,’” the report states.

“FDA confirmed that the total number of meetings between FDA staff and Biogen during this time is unknown because FDA lacked a ‘clear record’ of the informal meetings and other interactions between agency staff and Biogen,” the report notes, adding that of the more than 40 meetings held between the FDA and biogen, “not all were properly documented according to internal FDA procedures.”

FDA ‘Abruptly Changed Course and Granted Approval’

An additional 66 calls and substantive email exchanges between the agency and the company were also not documented, according to the report.

Elsewhere, the Committees said that FDA AND Biogen “inappropriately collaborated” for several months on a joint briefing document for the advisory committee’s review that didn’t adequately reflect differing views about the drug within FDA.

“For example, in an exchange of the draft briefing document on October 9, 2020, FDA staff asked Biogen to move a paragraph drafted by the agency into Biogen’s section of the memorandum—a change reflected when the document was finalized,” the report states. “FDA’s internal review determined that the Office of Neuroscience (ON) within CDER’s Office of New Drugs (OND) had failed to obtain internal OND consensus on FDA’s position prior to working with Biogen on the document.”

After considering Aduhelm under the traditional approval pathway for nine months, the investigation also found that the FDA “abruptly changed course and granted approval under the accelerated approval pathway—which allows the use of surrogate clinical endpoints to demonstrate effectiveness—after just three weeks of review.”

Finally, the report states that Biogen initially set an “unjustifiably high” price of $56,000 per year for Aduhelm in an effort to “make history” despite a lack of demonstrated clinical benefit and the anticipated financial impact on patients and the Medicare program.

The benefits of Aduhelm for each suitable patient are hard to predict. Jacob Lund/Shutterstock

Report ‘Raises Serious Concerns About FDA’s Lapses in Protocol’

“The findings in this report raise serious concerns about FDA’s lapses in protocol and Biogen’s disregard of efficacy and access in the approval process for Aduhelm,” the report concludes.

The committees recommended a number of steps be taken “help restore the American people’s trust” in the agency’s regulatory reviews, including ensuring that all substantive FDA interactions with drug companies are properly documented.

In a statement responding to the report, the FDA said its decision to approve Aduhelm was based on scientific evaluation of the data contained in the application.

“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,” the spokesperson said.

The agency will continue to use the accelerated approval pathway whenever appropriate but has already begun implementing some of the recommendations in the report, the spokesperson added.

Biogen said in a statement that it “stands by the integrity of the actions we have taken.”

More than six million people in the United States live with Alzheimer’s disease, according to the Alzheimer’s Association, although that number is expected to rise to as many as 14 million people by 2060. The disease kills more people than breast cancer and prostate cancer combined.

The Epoch Times has contacted The FDA and Biogen for comment.

Reuters contributed to this report.