Congressional report: U.S. FDA broke own protocols in approving Biogen Alzheimer’s drug

WASHINGTON, Dec 29 (Reuters) – The U.S. Food and Drug Administration failed to adhere to its own internal guidelines and practices during the approval process for Biogen’s ( BIIB.O ) Alzheimer’s drug Aduhelm, which was “riddled with irregularities,” a congressional report. shown on Thursday.

The FDA’s interactions with Biogen were “atypical” and did not follow the agency’s filing protocol, according to a staff report on the findings of an 18-month investigation conducted by two House committees into the drug’s regulatory review, approval, pricing and marketing.

The FDA approved Aduhelm in June 2021 under an accelerated approval pathway over the objections of its panel of outside advisors, who did not believe the data conclusively proved the drug’s benefit to patients.

It was authorized based on evidence that it could reduce brain plaques, a possible contributor to Alzheimer’s, rather than evidence that it slowed the progression of the deadly, mind-destroying disease.

The Medicare program limited its coverage, which has led to limited use of the Biogen drug.

Biogen set an “unjustifiably high” price by initially pricing Aduhelm at $56,000 a year despite a lack of demonstrated clinical benefit in a broad patient population, the report said, adding that the company’s internal projections showed that expected the medicine to be a burden. for Medicare and costly for patients.

“The findings in this report raise serious concerns about FDA’s protocol errors and Biogen’s disregard for efficiency and access in the approval process for Aduhelm,” the report, prepared by staff of the House Committee on Oversight and Reform and the House Committee on Energy and Commerce, concluded.

The agency should ensure that all essential interactions with drug sponsors are properly memorialized, establish a protocol for joint information documents with drug sponsors, and update its industry guidance on new Alzheimer’s drug developments and reviews, the report recommended.

Biogen and other drugmakers should communicate any safety and efficacy concerns to the FDA, and consider value and patient access when setting prices, the report said.

An FDA spokesman said the FDA’s decision to approve Aduhelm was based on scientific evaluation of the data included in the application.

He pointed to the FDA’s internal review finding its staff’s interactions with Biogen appropriate.

“It is incumbent on the agency to interact frequently with companies 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,” he said, adding that the agency will continue to use the expedited approval route whenever appropriate.

The FDA has already begun implementing some of the report’s recommendations, the spokesman said.

“Biogen stands by the integrity of the actions we’ve taken,” the Cambridge, Mass.-based biotech company said. in an emailed statement.

“As stated in the congressional report, an (FDA) review concluded that, “There is no evidence that these pre-filing interactions with the sponsor were anything but appropriate in this situation,” Biogen said.

Documents obtained by the committees show that FDA and Biogen staff held at least 115 meetings, calls and email exchanges over a 12-month period beginning in July 2019.

The total number of meetings is unknown because the FDA failed to keep a clear record of meetings and informal interactions between its staff and Biogen representatives. The investigation identified 66 other phone calls and email exchanges that were not memorialized.

FDA inappropriately collaborated with Biogen on a joint briefing document for the Peripheral and Central Nervous System (PCNS) Advisory Committee, the report said, with FDA and Biogen staff working closely for months before the November 6, 2020 meeting to prepare the document. , which failed to adequately represent the diverse views within the agency.

“Using a joint briefing document provided Biogen with advance knowledge of FDA responses and direct guidance from the agency in drafting the company’s own sections. For example, in an exchange of the draft briefing document on October 9, 2020, the staff of FDA asked Biogen to move an agency-drafted paragraph to Biogen’s section of the memo — a change reflected when the document was finalized,” the report released to media organizations said.

When none of the advisory panel members voted to approve Aduhelm, the FDA focused on using the expedited approval route — typically used for rare diseases or small patient populations that lack access to effective treatments — despite having considered the drug according to the traditional route of approval for nine. month, the report says.

It did so in a substantially shortened timeframe, approving it after three weeks of review and for a broad indication of the label “people with Alzheimer’s disease” that was not supported by clinical data, the report said.

Internal documents obtained by the investigation showed that Biogen accepted the indication despite its own reservations about the lack of evidence that Aduhelm could help patients in stages of the disease outside of clinical trials.

Reporting by Ahmed Abulenein; editing by Diane Craft

Our Standards: The Thomson Reuters Trust Principles.

Ahmed Abulenein

Thomson Reuters

Washington-based correspondent covering US health care and pharmaceutical policy with a focus on the Department of Health and Human Services and the agencies it oversees, such as the Food and Drug Administration, formerly based in Iraq and Egypt.