A study observed significant demographic and geographic disparities among participants of pivotal clinical trials that result in US Food and Drug Administration (FDA) approvals for leukemia and multiple myeloma (MM) treatments.
Outcomes for patients with hematologic malignancies have improved, but disparities in outcomes persist. Researchers sought to determine whether pivotal clinical trials of drugs approved for MM and leukemia are representative of the real-world patient population.
The researchers identified appropriate clinical trials via the FDA database and collected demographic and geographic information from ClinicalTrials.gov and the primary trial manuscripts.
Of the trials assessed, 41 (67.2%) reported data on race and 20 (48.8%) on ethnicity. These trials included 13,731 patients, most of whom were White (n=11,209; 81.6%).
Asian-Pacific Islander and Black patients had the highest representation in chronic myeloid leukemia (n=147, 12.7% and n=61, 5.3%, respectively) and the lowest representation in chronic lymphocytic leukemia (n=55, 3% and n=20, 1.1%, respectively).
The proportion of Black, Native American, and Hispanic trial participants was significantly low, reflecting underrepresentation in trials compared with the general population.
The study also found that women were underrepresented in acute myeloid leukemia (44.7% vs 60.5%; P<.0001), while men were underrepresented in MM (55.3% vs 60.2%; P<.0001) and chronic myeloid leukemia (55.2% vs 62.9%; P<.0001). The geographic distribution of study sites is not representative of the geographic distribution by mortality for all malignancies studied other than MM, according to the researchers.
The outcomes of this study suggest that data and understanding generated from these clinical trials are not representative of the general population, which may contribute to the disparities in cancer outcomes.
“These disparities need to be addressed to make results applicable to all relevant populations,” the researchers concluded.
Source: Journal of Clinical Oncology
