FDA-AACR Workshop:
Approach to Novel Oncology Endpoint Development
September 11, 2025
Alexandria, Virginia
workshop co-chairs
Lillian L. Siu, MD, FAACR, 2025-2026 President, American Association for Cancer Research
Nicole Gormley, MD, Division Director, Division of Hematologic Malignancies 2, Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, Maryland
Alex Snyder, MD, Co-Chair, Society for Immunotherapy of Cancer Biomarker Working Group
Overview
The U.S. Food and Drug Administration (FDA) Oncology Center of Excellence and the American Association for Cancer Research (AACR) collaborated to host 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 advanced the conversation around novel oncology endpoints by fostering shared learning between different disease areas and exploring opportunities to streamline the development of future endpoints.
program
Welcome and Session 1: Case Studies—FDA Experience with Novel Oncology Endpoint Development
- Welcome and introduction
Lillian L. Siu, MD, FAACR, Princess Margaret Cancer Center, Toronto Ontario, Canada
Alex Snyder, MD, Merck, New York, New York
Richard Pazdur, MD, U.S. Food and Drug Administration, Silver Spring, Maryland - Workshop overview and topic introduction
Nicole Gormley, MD, U.S. Food and Drug Administration, Silver Spring, Maryland - Minimal residual disease as an endpoint in multiple myeloma
Andrea Baines, MD, PhD, U.S. Food and Drug Administration, Silver Spring, Maryland - Lessons learned from pathological complete response as an endpoint in early breast cancer
Tatiana Prowell, MD, U.S. Food and Drug Administration, Silver Spring, Maryland
Session 2: Methodology and Body of Evidence to Support Use of a Novel Endpoint
Moderators: Pallavi Mishra-Kalyani, PhD, U.S. Food and Drug Administration, Silver Spring, Maryland; Qian Shi, PhD, Mayo Clinic, Rochester, Minnesota
- Statistical methodology for endpoint validation
Qian Shi, PhD, Mayo Clinic, Rochester, Minnesota
Session 3: Evidence Gaps in Endpoint Development—Case Studies in ctDNA
Moderator: Lillian L. Siu, MD, FAACR, Princess Margaret Cancer Center, Toronto Ontario, Canada
- ctDNA in colon cancer
Vaibhav Kumar, MD, U.S. Food and Drug Administration, Silver Spring, Maryland - ctDNA in lung cancer: FDA perspective
Paz Vellanki, MD, PhD, U.S. Food and Drug Administration, Silver Spring, Maryland - Toward incorporating MRD as an endpoint in lymphoma clinical trials
Ash Alizadeh, MD, PhD, Stanford University, Stanford, California
Session 4: Balancing the RIsks with the Potential Benefits of Use of a Novel Endpoint
Moderators: Alex Snyder, MD, Merck, New York, New York; Christopher Lieu, MD, University of Colorado, Aurora, Colorado
- Use of novel endpoints: Balancing benefits and risks
Tatiana Prowell, MD, U.S. Food and Drug Administration, Silver Spring, Maryland
Acknowledgements
We thank the Society for Immunotherapy of Cancer (SITC) for their invaluable input towards the organization and planning of this workshop.
Supporters
