The Food and Drug Administration’s approval process of Alzheimer’s drug Aduhelm has been deemed “rife with irregularities,” according to a congressional investigation released this week.
The report published Thursday detailed findings of an 18-month probe by the U.S. House of Representatives into the drug, its developer biotechnology company Biogen and FDA regulators.
Investigators found “atypical collaboration” between the company and the FDA, saying the federal agency went against its own protocols. The scathing report also criticizes Biogen's high price for the drug, saying the unjustifiably high cost impedes patient access.
Aduhelm, which became the first new FDA-approved drug since 2003, targets amyloid beta plaque, whose build-up is thought to play a role in Alzheimer’s. The accelerated approval last summer was controversial because the FDA sidestepped expert outside advisers who said clinical trials failed to prove effectiveness of the drug.
Two years before the approval, Biogen halted its trials early after mixed results that suggested the drug didn’t slow Alzheimer’s progression. Together, Biogen and the FDA began reanalyzing the data toward a conclusion that the drug works, ultimately leading to the FDA's conditional approval.
Federal report on FDA's approval of Aduhelm: What to know
Documents show "the agency’s review and approval process for Aduhelm was highly atypical and deviated from FDA’s guidance and procedures in significant respects,” the report by the House Oversight and Reform and Energy and Commerce committees reads.
The committees also said the FDA didn't properly document communication with Biogen.
According to the House report:
The FDA and Biogen collaborated “inappropriately” on a briefing document for a federal drug advisory committee that didn’t reflect differing views within the FDA on the drug’s effectiveness.
Despite staff’s doubts on clinical benefits for patients with more advanced Alzheimer’s, Biogen went with a “broad label” that Aduhelm was appropriate for all “people with Alzheimer’s disease.”
The FDA approved the label, which was a “far broader” population than the company studied in its clinical trials.
And the approval was far faster than normal. After nine months of review under a traditional process, the agency "abruptly changed course," pivoted to an accelerated review process and issued approval just three weeks after review.
Backlash after Aduhelm's launch over broad label, high price
Biogen Alzheimer’s disease team leaders even expressed concern that the company could lose credibility for using such a broad label. The summer it was approved, Biogen and FDA walked back the label after fierce public criticism.
Cost was also significant part of the public backlash following the drug’s launch. The report said the price for Aduhelm – $56,000 per year – was “unjustifiably high,” and that Biogen tried to “make history” for the company despite lack of clinical benefit. The company said it wanted to “establish Aduhelm as one of the top pharmaceutical launches of all time.”
Biogen was also well aware the hefty price would be a burden on Medicare and costly to patients, the report found. “Analyses conducted by Biogen estimated that some Medicare patients could face out-of-pocket costs for Aduhelm of up to 20 percent of their income,” the report read.
Still, the company planned an “aggressive” marketing approach to target doctors and patients to maximize revenue.
How did the FDA and Biogen respond to the findings?
In statements released Thursday, the company and its regulator defended the process, but the FDA said it is “already starting” to implement changes recommended by the committees, according to a statement to The New York Times and other media outlets.
What's next: FDA set to review another Alzheimer's drug in January
The findings come as the federal agency is expected to release a decision on whether to approve a different Alzheimer’s drug next week called lecanemab, which is also developed by Biogen along with Eisai.
The congressional reviewers wrote that the FDA must take “swift action” to ensure future processes for reviewing Alzheimer’s treatments don’t cast doubts on the integrity of the FDA’s review.
Investigators urged the FDA to adhere to protocol going forward, and said drug sponsors need to consider patient access when setting prices, calling sky-high prices for drugs with “unproven benefit” an “abuse of the public trust.”
“The Committees urge FDA, Biogen, and other drug sponsors seeking to develop treatments for Alzheimer’s disease and other diseases to follow guidance and protocols, provide transparency into the drug evaluation process and drug pricing, and work to better ensure public trust in future drug approvals,” the report reads.
Contributing: Ken Alltucker, USA TODAY; The Associated Press
This article originally appeared on USA TODAY: FDA controversy over Biogen's Alzheimer's drug Aduhelm, explained