Review of the FDA's Accelerated Approval Pathway

The FDA recently approved Aduhelm (aducanumab) to treat patients with Alzheimer's disease using the accelerated approval pathway. The accelerated approval pathway allows the FDA to approve drugs that treat serious conditions and that fill an unmet medical need based on a surrogate endpoint, which is a marker that is thought to predict a clinical benefit. The FDA's approval of Aduhelm raised concerns due to alleged scientific disputes within the FDA, the advisory committee's vote against approval, allegations of an inappropriately close relationship between the FDA and the industry, and the FDA's use of the accelerated approval pathway. In response to these concerns, we will assess how the FDA implements the accelerated approval pathway. This will include reviewing interactions between the FDA and outside parties as well as other aspects of the process, such as deciding on this pathway and scientific disputes. We will review the FDA's relevant policies and procedures, determine compliance with them, and make appropriate findings and recommendations based on a sample of drugs approved using the accelerated approval pathway, which will include Aduhelm. We will not assess the scientific appropriateness of the FDA's approval of any of the drugs under review. This work may result in multiple reports.

Announced or Revised	Agency	Title	Component	Report Number(s)	Expected Issue Date (FY)
Completed (partial)	Food and Drug Administration	Review of the FDA's Accelerated Approval Pathway	Office of Evaluation and Inspections	OEI-01-21-00400; OEI-01-21-00401	2025