Illustration: Shoshana Gordon/Axios
An 18-month congressional investigation found the Food and Drug Administration didn't follow its own guidance and inappropriately collaborated with drugmaker Biogen before the approval of a controversial Alzheimer's drug last year.
Why it matters: The findings, released in a report Thursday by the House Oversight and Energy and Commerce committees, come as the FDA reviews two other Alzheimer's drugs that target proteins believed to contribute to the development of Alzheimer's.
- While effective new treatments could deliver new hope to millions with the devastating condition, they also raise major questions about who'll pay the costs and how the U.S. will oversee what could become a multi-billion-dollar market.
Flashback: The FDA’s decision to approve Aduhelm was based on thin evidence, with the FDA's own staff saying it didn't meet the agency's customary standards.
- An outside advisory committee of experts recommended against approving the drug. Medicare later set limits on how it would cover Aduhelm, effectively dooming the drug's commercial prospects.
What they found: FDA staff and Biogen engaged in at least 115 meetings, calls, and substantive email exchanges surrounding the drug Aduhelm over a 12-month period beginning in July 2019 — interactions that were "atypical" and weren't all properly documented according to internal FDA procedures, per the report.
- FDA and Biogen inappropriately collaborated on a briefing document for the advisory committee that didn't adequately reflect differing views about the drug within FDA.
- After considering Aduhelm under the traditional approval pathway for nine months, the investigation found that the FDA abruptly pivoted and granted approval through a fast-track process that lets patients access new drugs before there are full, real-world clinical results.
- Biogen initially set Aduhelm’s price at an "unjustifiably high" $56,000 per year despite a lack of demonstrated clinical benefit, the anticipated financial impact on patients and the Medicare program, in order to maximize revenue and "make history."
What they're saying: The Democratic-led committees recommended corrective steps to regulatory reviews, such as ensuring that all substantive FDA interactions with drug companies are properly documented and establishing a protocol for joint FDA-drug sponsor briefing documents for advisory committees.
- The FDA said it fully cooperated with the investigation, adding, "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 it has started making changes consistent with the committees' findings, including examining the use of a joint briefing document and plans to update its guidance to drugmakers on the development and review of new Alzheimer's drugs.
- Biogen said in a statement that it stands by the integrity of the actions it took.