The Food and Drug Administration’s (FDA) process for approving a controversial Alzheimer’s drug with questionable efficacy was “atypical” and “rife with irregularities,” according to a report released Thursday by two House committees.
According to the joint report from the Energy and Commerce and Oversight and Reform panels, FDA and Aduhelm manufacturer Biogen inappropriately collaborated and failed to follow the agency’s process for documenting the meetings.
The agency then allowed Aduhelm to be granted accelerated approval, after which it entered the market at a cost of $56,000 a year.
The findings “raise serious concerns about FDA’s lapses in protocol and Biogen’s disregard of efficacy and access in the approval process for Aduhelm,” according to the report.
“The American people rely on FDA for assurance on the safety and efficacy of the medications they take, and it is incumbent upon drug companies such as Biogen to ensure that the well-being and safety of patients are prioritized,” the House lawmakers added.
In a statement, Oversight Committee Chairwoman Carolyn Maloney (D-N.Y.) said she hopes the findings “are a wake-up call for FDA to reform its practices and a call to action to my Congressional colleagues to continue oversight of the pharmaceutical industry to ensure they don’t put profits over patients.”
Internal documents obtained by the committees showed 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, including at least 66 calls and substantive email exchanges that weren’t properly documented.
FDA guidance says that the agency is supposed to document all “substantive” communications between the agency and drug sponsors.
Biogen then worked with FDA staff on a joint briefing document ahead of a key agency advisory panel meeting in November 2020, which congressional investigators said afforded Biogen “advance insight into FDA’s responses and direct guidance from the agency.”
Agency staff and the company worked so closely together that it made “distinguishing between the agency’s and the sponsor’s respective analyses and positions challenging,” the report said.
In a statement, the FDA said it “fully cooperated with the Committees’ evaluation and we continue to review their findings and recommendations.”
“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 FDA said. “That said, the agency has already started implementing changes consistent with the Committee’s recommendations.”
The FDA generally follows an advisory committee’s recommendation, although it is not required to do so. None of the 11 members of the committee recommended Aduhelm approval, but the FDA suddenly decided to consider it under the accelerated approval pathway.
Despite internal concerns raised by experts about the inconsistency of the drug’s clinical data, the agency approved Aduhelm in early June 2021. The FDA also gave the drug an unexpectedly broad label, saying it could be used on all Alzheimer’s patients regardless of severity, even though it had been tested only on people with early Alzheimer’s and mild symptoms.
Biogen’s own leaders and outside stakeholders raised concerns over such a broad label, but did not ultimately object, the report found.
“This report documents the atypical FDA review process and corporate greed that preceded FDA’s controversial decision to grant accelerated approval to Aduhelm,” Energy and Commerce Chairman Frank Pallone, Jr. (D-N.J.) said in a statement.
According to the report, Biogen knew its $56,000 launch price was “unjustifiably high,” but company executives wanted to “make history” and “establish Aduhelm as one of the top pharmaceutical launches of all time.” The company estimated a potential peak revenue of $18 billion per year.
Documents provided to the committees showed that Biogen fully expected the high price would spur “pushback” from providers and payers, and that the company knew the price would be a burden to Medicare and patients. An internal presentation showed estimates the drug could cost Medicare more than $12 billion a year.
In anticipation of the backlash, Biogen developed an external narrative about the drug’s value to sell to patients and the public. In some long-range plans, Biogen anticipated spending more than $3.3 billion on sales and marketing for Aduhelm from 2020 to 2024, the report found.
In January, Biogen dropped the price of Aduhelm to $28,000 a year. Even so, its pricing strategy backfired — the drug made only $3 million in 2021 amid the backlash.
In a statement, Biogen said it “stands by the integrity of the actions we have taken” and that “we have learned from the development and launch of Aduhelm.”
The company also noted that the FDA conducted its own internal investigation more than a year ago, which concluded there was “no evidence” that the interactions between the agency and the company in advance of filing “were anything but appropriate in this situation.”
The FDA’s internal investigation had not been previously disclosed by the agency. According to the congressional report, the agency said the extended collaboration with Biogen “exceeded the norm in some respects.”