Demographic Trends in NCI-sponsored Early-phase Clinical Trials (2000-2023): A Cohort Study

We analyzed demographic trends among patients enrolled in NCI Cancer Therapy Evaluation Program-sponsored early-phase clinical trials during 2000 to 2023. Using a large patient subset (N = 16,609; 15,308 U.S. residents), we assessed enrollment trends via 3-year rolling averages and Joinpoint analysis. U.S. residents were matched to census demographics, rurality, geographic, and poverty data by ZIP code and Surveillance, Epidemiology, and End Results 2022 cancer incidence rates. Enrollment increased among patients aged 65 to 74 (24.6%-32.3%) and 75 to 84 (6.2%-12.4%) and among Hispanic/Latino (4.5%-7.7%), Asian/Pacific Islander (2.2%-4.4%), and non-Hispanic Black (6.1%-7.2%) patients. However, participation remained below cancer incidence rates for Hispanic/Latino and non-Hispanic Black patients and was negligible among American Indian and Alaska Native populations. Representation improved significantly following the 2014 implementation of the Experimental Therapeutics Clinical Trials Network, accompanied by a reduction in the average distance between patients' residences and enrolling institutions.

This is the first study that evaluates the demographic representation trends in early-phase clinical trials spanning over two decades. It provides valuable insights to help broaden participation in future trials and inform meaningful policy changes to address persistent gaps in oncology research and clinical care. See related commentary by Hughes Halbert, p. 2194.