FDA-AACR Workshop:
Approach to Novel Oncology Endpoint Development
Date: September 11, 2025 | 8:30 a.m.-3:30 p.m. ET
Location: Hilton Alexandria Old Town, 1767 King St, Alexandria, Virginia, and online
**See below for recordings and slides**
Workshop Agenda Speaker Bios Workshop Summary BlogOverview
The U.S. Food and Drug Administration (FDA) Oncology Center of Excellence and the American Association for Cancer Research (AACR) are collaborating on a one-day hybrid workshop to explore considerations for the development and application of novel endpoints in oncology drug development. This effort builds on the 2023 FDA-AACR-ASA workshop on overall survival (OS) as a clinical trial endpoint.
Although OS remains an important safety and efficacy measurement, it is not always feasible to use OS as the primary endpoint in interventional clinical trials in oncology, particularly in early-stage disease and rare cancer settings. To address this, there have been several novel endpoints developed by the broader community that are able to inform regulatory decisions. Recent examples include pathologic complete response in breast cancer and minimal residual disease in multiple myeloma. However, the use of novel endpoints carries inherent risks. Use of an endpoint that is not reflective of long-term clinical benefit could result in the approval of a product which is not safe and effective. This could lead to patients forgoing therapies that could extend their overall survival, while they instead receive an ineffective therapy. As such, rigorous characterization of novel endpoints, including establishing their relationship to long-term outcomes, is essential.
This workshop aims to advance the conversation around novel oncology endpoints by fostering shared learning between different disease areas and exploring opportunities to streamline the development of future endpoints. Sessions will explore topics including previous successful endpoint development approaches, areas of greatest unmet need for further development, and the evidence needed to support the use of novel endpoints in regulatory decision-making, particularly within the context of accelerated approval pathways.
workshop co-chairs
Lillian L. Siu, MD, President, American Association for Cancer Research
Nicole Gormley, MD, Division Director, Division of Hematologic Malignancies 2, Oncology Center of Excellence, U.S. Food & Drug Administration
Alex Snyder, MD, Co-Chair of Society for Immunotherapy of Cancer Biomarker Working Group
program
Welcome and Session 1: Case Studies: FDA Experience with Novel Oncology Endpoint Development
Session 2: Methodology and Body of Evidence to Support Use of a Novel Endpoint
Session 3: Evidence Gaps in Endpoint Development: Case Studies in ctDNA
Session 4: Balancing the Rusks with the Potential Benefits of Use of a Novel Endpoint and Closing Remarks
Acknowledgements
We thank the Society for Immunotherapy of Cancer (SITC) for their invaluable input towards the organization and planning of this workshop.
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