In a study reported in the Journal of Clinical Oncology, Casey et al found that most U.S. minority populations were underrepresented in clinical trials leading to U.S. Food and Drug Administration approval of drugs for treating leukemias and multiple myeloma.
Study Details
The investigators identified 61 trials leading to approvals for leukemias and multiple myeloma between January 1, 2011, and October 1, 2021. Of these, 41 (67.2%) reported data on race and 20 (48.8%) reported data on ethnicity.
Key Findings
The 41 trials reporting data on race included 13,731 patients, of whom 11,209 (81.6%) were White.
In trials in multiple myeloma, White, Black, Asian/Pacific Islander, Native American, and Hispanic patients accounted for 80.3%, 4.7%, 10.7%, < 0.1%, and 2.9% of the population; by comparison, Surveillance, Epidemiology, and End Results (SEER) 2018 data estimates show a disease distribution of 68.7%, 27.4%, 3.4%, 0.2%, and 17.5%, respectively.
In trials in acute myeloid leukemia, White, Black, Asian/Pacific Islander, Native American, and Hispanic patients accounted for 79.6%, 3.8%, 8.8%, 0.1%, and 6.3% of the population; by comparison, SEER estimates show a disease distribution of 77.3%, 12.3%, 5.6%, 1.5%, and 15.3%, respectively.
In trials in chronic myeloid leukemia, White, Black, Asian/Pacific Islander, Native American, and Hispanic patients accounted for 76.7%, 5.3%, 12.7%, 0.2%, and 7.6% of the population; by comparison, SEER estimates show a disease distribution of 75.5%, 13.2%, 4.2%, < 0.1%, and 13.8%, respectively.
In trials in acute lymphoblastic leukemia, White, Black, Asian/Pacific Islander, Native American, and Hispanic patients accounted for 79.2%, 2.2%, 7.2%, 0.8%, and 11.5% of the population; by comparison, SEER estimates show a disease distribution of 79.7%, 7.8%, 6.1%, 1.6%, and 25.7%, respectively.
Females were underrepresented in trials in acute myeloid leukemia (44.7% vs 60.5% in SEER data) and males were underrepresented in trials in multiple myeloma (55.3% vs 60.2%) and chronic myeloid leukemia (55.2% vs 62.9%).
The geographic distribution of trials showed inadequate regional and state participation compared with mortality for all malignancies except multiple myeloma.
The investigators concluded, “There are significant demographic and geographic underrepresentation and imbalances in pivotal clinical trials leading to drug approvals for leukemias and multiple myeloma compared with the population affected. These disparities need to be addressed to make results applicable to all relevant populations.”
Jorge E. Cortes, MD, of the Georgia Cancer Center at Augusta University, is the corresponding author for the Journal of Clinical Oncology article.
Disclosure: For full disclosures of the study authors, visit ascopubs.org.
