The US Food and Drug Administration (FDA) initiated the accelerated approval pathway in 1992 in response to the HIV epidemic as a way to expedite clinical trial evaluations of promising drugs in the treatment of serious or life-threatening disease. The accelerated approval pathway explicitly allows biomarkers to be used as primary efficacy end points and permits those biomarkers to be only “reasonably likely” to predict clinical benefit. When a drug receives accelerated approval, there is usually lesser certainty of clinical benefit than if approval were based on clinical outcomes, such as improvements in how a patient feels or functions or prolonged survival. In contrast, the FDA’s regular approval pathway is typically based on clinical outcomes, although in recent years the agency has permitted the use of surrogate markers for clinical benefit with increasing frequency.1
Identify all potential conflicts of interest that might be relevant to your comment.
Conflicts of interest comprise financial interests, activities, and relationships within the past 3 years including but not limited to employment, affiliation, grants or funding, consultancies, honoraria or payment, speaker's bureaus, stock ownership or options, expert testimony, royalties, donation of medical equipment, or patents planned, pending, or issued.
Err on the side of full disclosure.
If you have no conflicts of interest, check "No potential conflicts of interest" in the box below. The information will be posted with your response.
Not all submitted comments are published. Please see our commenting policy for details.
Gyawali B, Ross JS, Kesselheim AS. Fulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway: The Need for Reforms. JAMA Intern Med. Published online July 13, 2021. doi:10.1001/jamainternmed.2021.4604
Coronavirus Resource Center
Customize your JAMA Network experience by selecting one or more topics from the list below.
Create a personal account or sign in to: