Phase 1 Oncology Trials: Improved Outcomes Over Time

Overall response rates in these trials nearly doubled from 2000 to 2019, without an increase in treatment-related death.

Phase 1 trials are frequently viewed as having a low probability of benefiting an individual patient, despite the novelty of agents being investigated. Often forgotten is that these trials are primarily designed to define the optimal dose of a drug to take forward for further development. A secondary goal is to identify signals of antitumor activity. Although the fraction of novel agents that go on to FDA-approval remains modest, the collective progress made with phase 1 trials deserves re-evaluation.

Investigators analyzed patient-level data from the Cancer Therapy Evaluation Program of the National Cancer Institute–sponsored investigator-initiated phase 1 trials for solid tumors between 2000 and 2019, with particular attention to high-grad…

Reviewing Author

William J. Gradishar, MD

Disclosures

Consultant/Advisory BoardLilly; AstraZeneca; Gilead

Grant/Research SupportBreast Cancer Research Foundation

Editorial BoardsClinical Breast Cancer; Oncology; Annals of Surgery; Breast Cancer Research and Treatment

Leadership Positions in Professional SocietiesNational Comprehensive Cancer Network (Chair, Breast Cancer Panel); American Board of Internal Medicine (Medical Oncology Board)