Today, Oversight and Reform Committee Chairwoman Carolyn B. Maloney (D-NY) and Energy and Commerce Committee Chairman Frank Pallone, Jr. (D-NJ) released a staff report following an 18-month investigation into the Food and Drug Administration’s (FDA) regulatory review and approval process of Biogen’s Alzheimer’s disease drug Aduhelm, and Biogen’s pricing of Aduhelm.
“One of my top priorities as Chair of the Committee on Oversight and Reform is ensuring that the American people have access to effective and affordable medications," Chairwoman Maloney said. “The number of patients and families impacted by Alzheimer’s disease will continue to increase, and it is crucial that FDA and drug companies adhere to established procedures and conduct themselves with the transparency necessary to earn public trust. I am hopeful these 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."
“This report documents the atypical FDA review process and corporate greed that preceded FDA’s controversial decision to grant accelerated approval to Aduhelm." Chairman Pallone said. “While we all support the search for new cures and treatments to address devastating diseases like Alzheimer’s, we must ensure that expediency does not take precedence over protocols that ensure the independence and scientific rigor of FDA. Patient safety and drug efficacy must remain at the core of our nation’s pharmaceutical regulatory review process. FDA must continue to take corrective actions to re-earn the trust of the American people, and Biogen and other pharmaceutical manufacturers must also learn from the issues outlined in this report and implement our recommendations to place the well-being of patients over profits."
The report details how, leading up to November 2020, FDA and Biogen prepared and presented a joint briefing document on Aduhelm to a key FDA advisory committee. Following the presentation, none of the committee members voted to recommend traditional approval for Aduhelm. After the advisory committee meeting, Public Citizen, a non-profit consumer rights advocacy group, wrote to FDA expressing concern over alleged close collaboration between the agency and Biogen. In response, FDA conducted an internal review of the agency and Biogen’s interactions prior to the November 2020 advisory committee meeting. The previously unreleased internal FDA review, which was completed in May 2021, was released today with the Committees’ report.
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. Later that month, Chairs Maloney and Pallone announced the Committees’ investigation into FDA’s decision to grant accelerated approval to Aduhelm.
The Committees’ report has uncovered the following information:
* FDA’s interactions with Biogen were atypical and failed to follow the agency’s own documentation 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. Despite FDA guidance that substantive interactions between agency staff and drug sponsors should be documented, the agency confirmed the total number of meetings between agency 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 Committees’ investigation identified an additional 66 calls and substantive email exchanges among the subgroups of the working group that were not memorialized.
* FDA and Biogen inappropriately collaborated on a joint briefing document for a key advisory committee. The Committees obtained evidence that FDA and Biogen staff worked closely for several months ahead of the November 6, 2020, Peripheral and Central Nervous System Drugs Advisory Committee meeting to prepare the joint briefing document for the committee’s review. Documents show that using a joint briefing document afforded Biogen advance insight into FDA’s responses and direct guidance from the agency in drafting the company’s own sections, including sections drafted by FDA that were then included in Biogen’s portion of the document. In addition, the document did not adequately represent differing views, and FDA’s own internal review concluded that “the use of the joint briefing document was not an appropriate approach in this instance" given the substantial disagreement among FDA offices.
* FDA pivoted to using the accelerated approval pathway for Aduhelm on a substantially abbreviated timeline. Documents and information obtained by the Committees show that FDA considered Aduhelm under the traditional approval pathway used for most drugs for nine months, before abruptly changing course and granting approval under the accelerated approval pathway after a three-week review period.
* FDA approved and Biogen accepted a broad label indication for Aduhelm, despite lack of clinical data on all Alzheimer’s disease stages and Biogen’s reservations. Materials obtained by the Committees demonstrate that FDA recommended and approved a broad label indication, despite the 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, and an unknown safety profile.
* Biogen initially set an unjustifiably high price for Aduhelm, at $56,000 per year, to “make history" for the company, despite the impact on patients and the Medicare program. 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. A September 2020 presentation to the Board stated, “Our ambition is to make history" and “establish ADUHELM as one of the top pharmaceutical launches of all time." Biogen received a report from third-party consultants, who provided strategic guidance on pricing Aduhelm, which 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.
* Biogen expected Aduhelm to be a burden to Medicare and costly to patients. Internal company documents show that Biogen was aware the financial burden of its high price for Aduhelm would fall primarily on Medicare. Biogen estimated that the drug would cost Medicare $12 billion in one year-representing 36 percent 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.
* Biogen planned to spend billions to market Aduhelm, despite the financial impact on patients and the health care system. Internal company documents show that Biogen planned an aggressive outreach and marketing campaign to launch Aduhelm, focusing on direct outreach to providers, patients, patient advocacy groups, payers, and even policymakers. In some long-range plans, Biogen anticipated spending more than $3.3 billion on sales and marketing for Aduhelm from 2020 to 2024-more than two and a half times what Biogen spent in total development costs for aducanumab from 2007 until approval in June 2021.
Based on the investigation’s findings, the Committees’ report makes several recommendations to FDA intended to help restore the American people’s trust in the agency’s processes and assurances of drug safety and efficacy. The report also recommends actions that Biogen and other drug sponsors take in the future to fulfill their responsibility to the patients and families who rely on their treatments.
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