Exploring Novel Oncology Endpoints

“While overall survival remains the gold standard endpoint, it becomes challenging [to measure] in clinical trials… [which can] take many years to read out. To expedite drug development and move promising treatments to patients faster, we are hoping that we can identify and validate novel endpoints that allow us to measure a treatment’s efficacy much earlier than we can with overall survival.”

The opening remarks of AACR President (2025-2026) Lillian L. Siu, MD, FAACR, highlighted the purpose of a recent FDA-AACR Workshop on Approaches to Novel Oncology Endpoint Development. The workshop, held September 11 in Alexandria, VA, was co-chaired by Siu; Nicole Gormley, MD, associate director of Oncology Endpoint Development at the FDA Oncology Center of Excellence; and Alexandra Snyder, MD, cochair of the biomarkers committee of the Society for Immunotherapy of Cancer (SITC).

The workshop highlighted several early endpoints currently in regulatory use—including pathologic complete response and progression free survival—and outlined pathways to streamline the development of novel endpoints that may serve as early indicators of clinical benefit, such as minimal residual disease measured by circulating tumor DNA. Experts also weighed the risks of using novel early endpoints against the potential benefit of faster patient access to effective treatments, and identified settings where the benefits and risks would be best balanced.