WASHINGTON — A joint investigation released Thursday by House Democrats identified unusual collaboration between Food and Drug Administration officials and drugmaker Biogen, highlighting potential problems in the agency’s approval of the controversial Alzheimer’s drug Aduhelm.
The House Energy and Commerce and Oversight and Reform committees concluded their findings “raise serious concerns” about the drug’s approval process.
“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,” Oversight and Reform Chair Carolyn B. Maloney, D-N.Y., said in a statement, calling the report a “wake-up call” for the FDA.
The FDA previously determined in an internal review that officials acted appropriately, but said it has already begun implementing the report’s 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,” the agency said in a statement. “We will continue to do so, as it is in the best interest of patients.”
Biogen refuted the report’s implications, saying it “stands by the integrity of the actions we have 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,’” the company said.
The Federal Trade Commission and Securities and Exchange Commission also opened investigations into Biogen following the drug’s approval.
The FDA approved the drug in June 2021 on the condition that it receive additional follow-up data, but concerns over the drug’s efficacy and safety profile led the Centers for Medicare and Medicaid Services to restrict coverage of it and related drugs authorized through the FDA’s accelerated approval process to clinical trials.
The decision doomed the drug’s market potential and cast doubt on other monoclonal antibody drugs targeting brain plaques in Alzheimer’s patients. The FDA is also currently reviewing a similar drug, lecanemab, from Biogen and its Japan-based partner, Eisai. A decision on accelerated approval is due by Jan. 6, and the company is simultaneously submitting data for full approval based on results from a Phase 3 trial.
The congressional report offers the latest lens into the Aduhelm’s saga, which began after Biogen halted clinical trials over disappointing results in 2019. But the company reversed course months later, arguing that a new analysis revealed the drug’s potential benefit.
The report labeled the FDA’s subsequent actions as “atypical” and “rife with irregularities,” pointing to undocumented interactions with Biogen and the preparation of an unusual joint briefing document for a key advisory committee.
That panel, the Peripheral and Central Nervous System Drugs Advisory Committee, recommended the FDA reject the drug. But the agency suddenly okayed Aduhelm through the accelerated approval pathway — an option that was not presented to committee members. Several resigned in protest.
The report also found the FDA initially granted Aduhelm a broad label for all Alzheimer’s patients, in spite of Biogen’s reservations over a lack of data on late-stage patients. But Biogen did not push back, and later requested the label be narrowed to just early-stage cases after public backlash.
Experts also raised concerns about the drug’s potential to drain Medicare’s finances in the wake of its approval, thanks to its high price tag. Internal documents obtained by the committees show Biogen calculated maximum patient access at roughly one-third of its initial $56,000 list price. The company also planned a four-year, $3.3 billion marketing strategy.
The report recommended that FDA improve documentation of meetings and clarify proper protocols — such as collaborating with drugmakers on briefing documents — to avoid future controversies.
“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 said, also citing FDA’s pending review of lecanemab.
“The criticism surrounding Aduhelm’s approval may have been avoided had FDA adhered to its own guidance and internal practices,” the report continued. “FDA must take swift action to ensure that its processes for reviewing future Alzheimer’s disease treatments do not lead to the same doubts about the integrity of FDA’s review.”
