U.S. health officials’ approval process for Biogen Inc.’s controversial Alzheimer’s drug was “rife with irregularities,” raising serious concerns about protocol lapses at the agency, congressional investigators said.
The Food and Drug Administration collaborated excessively with Biogen while assessing the drug, called Aduhelm, according to the report on an 18-month investigation published Thursday by two House committees, Oversight and Reform and Energy and Commerce.
Aduhelm was cleared despite objections from a group of outside medical experts who advised the FDA not to allow the drug on the market, citing conflicting results from trials of its efficacy; three members of that panel resigned after the approval. The U.S. Medicare program later heavily restricted payment for Aduhelm, and Biogen stopped its marketing efforts.
The report “describes a pattern of incredibly troubling behavior around the approval of this drug,” said Aaron Kesselheim, a professor at Harvard Medical School who left the advisory panel after Aduhelm’s approval. “The FDA is the world’s most important public health regulator, but when it takes steps like this, it damages its own credibility and the trust that patients and physicians have in it.”
Aduhelm gained clearance in June 2021 not by showing effectiveness against brain-wasting, but its ability to reduce amyloid plaques in the brain, a physical marker linked to the disease. Another experimental drug that reduces amyloid, Eisai Co.’s lecanemab, which is being developed in collaboration with Biogen, returned positive results in slowing the disease in September.
Biogen said it has been committed to researching and developing treatments for Alzheimer’s disease for more than a decade, and that it stands by the integrity of its actions. The FDA said it remains committed to the integrity of its approval process and that its internal review found that the staff’s work with Biogen was appropriate.
“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 FDA said in a statement.
Biogen viewed Aduhelm as an “unprecedented financial opportunity,” according to the congressional report that criticized Biogen for setting an unjustifiably high price of $56,000 a year for the product. The company developed aggressive launch and marketing plans designed to maximize revenue, despite being aware that the treatment would be costly to patients and be a burden to Medicare, the report said.
Biogen has since cut the price of Aduhelm to $28,200 in order to lower out-of-pocket costs for patients and reduce “the potential financial implications for the U.S. health-care system.” However, this decision was made only after public backlash, the report said.
The FDA initially approved the treatment with a label allowing its use in a wide swath of Alzheimer’s patients, rather than the very early-stage patients the drug was mostly studied on. Even as the company accepted the broader use statement, there were internal concerns about lack of evidence to support it, the investigators found. Biogen only later sought to narrow the conditions for the drug’s use after public criticism, the report said.
Documents obtained by the congressional committees found that the FDA and Biogen held “at least 115 meetings, calls, and substantive email exchanges” during a 12-month period as Biogen worked to complete and submit its application for Aduhelm. That may have been an undercount, as the FDA didn’t have a clear record of its meetings with Biogen.
The FDA also used a controversial pathway, called accelerated approval, to assess Aduhelm. Critics have said that pathway allows many drugs to get to market on the basis of questionable data.
The FDA said it will continue using the accelerated pathway when appropriate, as it allows the agency to provide earlier access to serious, life-threatening conditions.
The report’s findings should serve as “a wake-up call for FDA to reform its practices,” Representative Carolyn Maloney of New York, chair of the Oversight and Reform Committee, said in a statement.
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