Clinical trials leading to the approval of drugs for patients with leukemia or multiple myeloma (MM) may not accurately represent the patient groups intended for treatment, according to research published in the Journal of Clinical Oncology.

It has repeatedly been shown that sociodemographic factors affect clinical outcomes across cancer subtypes, including in MM and leukemia. This may be in part due to access to care, which is lower among elderly and minority patients. As clinical trials (CTs) frequently require that enrolled patients be physically present, it is possible that these studies — upon which FDA approvals are based — are not sufficiently representative of the population groups they are designed to help.

While there have been policy attempts to improved representation in CTs, it is unknown the degree to which all patient subgroups are represented. For this study, researchers analyzed racial, ethnic, age, and sex data from trial participants in CTs upon which drug approval was based relative to the overall disease burden in leukemia and MM.

Continue Reading

The authors reviewed data from 61 CTs with information available on Forty-one trials leading to drug approval reported data on patient race; 20 reported data on patient ethnicity. Together, these CTs included data from 13,731 patients. Of these, 11,209 (81.6%) were White.

In studies into chronic myeloid leukemia, Asian-Pacific Islanders and Black patients were the best represented of minority groups (12.7% and 5.3%, respectively), although they were the worst represented in studies into chronic lymphocytic leukemia (3% and 1.1%, respectively). Overall, Black patients, Native Americans, and Hispanic patients were consistently under-represented in the analyzed clinical trials compared with the general population.

Female sex patients were under-represented in studies into acute myeloid leukemia (44.7% vs 60.5% for males; P <.0001), though male sex patients were under-represented in studies into MM and chronic myeloid leukemia.

Furthermore, aside from MM, all studies did not adequately include patients from different regions and states across the US.

“Trials should represent the population with the disease and should be within reach of patients in areas of need,” the authors wrote in their report. “These disparities within cancer research need to be addressed to make results applicable to all populations.”

Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 


Casey M, Odhiambo L, Aggarwal N, et al. Are pivotal clinical trials for drugs approved for leukemias and multiple myeloma representative of the population at risk? J Clin Oncol. 2022;40(32):3719-3729. doi:10.1200/JCO.22.00504