Navigating Recent Changes to FDA Guidance on Accelerated Approval: Key Considerations for Sponsors
Author:
Dr Robert Dow
Chief Medical Officer
Co-Authors:
Dr Rebbecca Wilcox
Principal Consultant
Dr Maria Beatrice Panico
Principal Medical Consultant
Ellie McNamee
Consultant
Introduction
The FDA accelerated approval pathway was established in 1992 (FDA, 1992) to expedite access to new therapies for patients with serious or life-threatening diseases. First created to rapidly address unmet medical need during the HIV-AIDS crisis, oncology drugs now comprise the largest proportion of accelerated approvals (Sachs et al., 2021). Use of this pathway allowed the FDA to grant approval sooner based on surrogate endpoints. These surrogates must be deemed reasonably likely to predict clinical benefit. However, the Sponsor has been historically required to conduct confirmatory trials verifying the clinical benefit, post granting of accelerated approval.
The FDA’s guidelines were updated in March 2023 (Benjamin et al., 2023). This paper discusses the other events and publications that had a significant influence on the emergence of the new guidelines, describes the major impact that the new guidelines will have on the development of oncology drugs, and discusses what information is available to date on their interpretation and impact in the two years since their publication. This paper also summarises additional guidelines on the subject of accelerated approval published in December 2024 (FDA, 2024a) and January 2025 (FDA, 2025a).