The Food and Drug Administration (FDA) has released a draft guidance to clarify the requirements for confirmatory trials for drugs granted accelerated approval. This guidance, titled "Accelerated Approval and Considerations for Determining Whether a Confirmatory Trial is Underway," outlines the FDA's interpretation of the term "underway" and details the factors the agency intends to consider when determining whether a confirmatory trial meets this requirement prior to granting accelerated approval.
The draft guidance is a response to amendments made to Section 506(c) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) by the Consolidated Appropriations Act of 2023 (CAA). These amendments grant the FDA additional authority to ensure the timely completion of confirmatory trials, which are required to verify and describe the anticipated effect of the drug on irreversible morbidity or mortality, or other clinical benefits.
The guidance specifies that the FDA may require a confirmatory study or studies to be underway before or within a specified period after the approval date of the product. This requirement aims to address concerns about delays in the completion of post-approval confirmatory trials, which are crucial for verifying the clinical benefit of drugs approved under the accelerated approval pathway.
The FDA will use postmarketing reports submitted by sponsors to monitor the progress of confirmatory trials and take action if necessary. These reports are required approximately every 180 days. The information is essential to support the FDA's efforts to protect the health of users of drugs approved under accelerated approval.
Stakeholders are invited to submit electronic or written comments on the draft guidance by March 10, 2025, to ensure the agency considers their input before finalizing the guidance. The FDA will consider all comments received and revise the guidance as appropriate.
The draft guidance is consistent with the FDA's good guidance practices regulation (21 CFR 10.115) and represents the agency's current thinking on the matter. It does not establish any rights for any person and is not binding on the FDA or the public. Alternative approaches that satisfy the requirements of applicable statutes and regulations may be used.
