The Commonwealth Fund: The Accelerated Approval Pathway for New Drug Therapies
In June 2021, the Food and Drug Administration (FDA) approved the Alzheimer’s drug Aduhelm through the agency’s Accelerated Approval Program. This pathway allows for earlier approval of drugs that offer a meaningful benefit over existing therapies and treat serious conditions that have an unmet medical need. Controversy surrounding Aduhelm’s extremely high cost, along with questions over its clinical benefit and health risks to patients, has placed the accelerated approval pathway under scrutiny.
How does the accelerated approval pathway work?
Established in 1992 in response to the HIV/AIDS crisis, the accelerated approval pathway allows the FDA to speed approval of drugs based on a “surrogate endpoint” rather than a direct measure of clinical benefit, as required for traditional FDA approval. Surrogate endpoints are substitute measures that are either known or likely to predict clinical benefit but do not directly measure clinical benefit. For example, the benefit of a drug submitted for accelerated approval might be determined through laboratory measurements or radiographic imaging rather than its ability to prolong life. Once approved via the accelerated approval pathway, the manufacturer must study the drug further to confirm its clinical benefit. If these postapproval conditions aren’t met, the FDA can withdraw its approval.