An evaluation of race and ethnicity-based representation among participants in phase III clinical trials for multiple myeloma

Background: The inclusion of racial/ethnic minorities in clinical trials is essential to fully assess the therapeutic safety and efficacy of a medical drug and/or device. However, clinical trials historically do not mirror patient population demographics.

Objectives: This study aimed to comprehensively characterize the representation of racial/ethnic minorities in clinical trials for multiple myeloma.

Methods: The disease keyword “multiple myeloma” was used to query on ClinicalTrials.gov with primary completion date of trials during 2016-2020 and with any US site location. A total of 28 phase III trials of multiple myeloma with available results were assessed, representing 14,927 participants.

Results: Race or ethnicity was reported in 23 trials (82.1%; 12,856 participants). Among the reported data, participants were overwhelmingly White (82.3%), with 4.4%, 9.9%, and 0.1% reported as Black/African American, Asian/Pacific Islander, and American Indian/Alaska Native, respectively. The percentages of non-White racial groups increased over time, from 15.5% for studies started before 2011 to 17.9% during 2011-2015 and 20.4% after 2015. Within the 19 trials (11,226 participants) reporting ethnicity, only 4.5% of participants were reported as Hispanic/Latino. Among six trials conducted exclusively in the US (1,747 participants), race distribution was reported in all trials, with 77.2%, 15.4%, 1.1%, and 0% reported as White, Black/African American, Asian/Pacific Islander, and American Indian/Alaska Native, respectively. The percentages for all non-White races among multiple myeloma US patients were lower than those reported in the National Cancer Institute Surveillance, Epidemiology, and End Result (SEER) data 2016-2020 (20.9%, 4.7%, and 0.3% for Black/African American, non-Hispanic Asian/Pacific Islander, and non-Hispanic American Indian/Alaska Native, respectively). Similarly, within three US trials with ethnic data, 5.7% of participants were reported as Hispanic/Latino, which was lower than the SEER data 2016-2020 (14.3%).

Conclusions: Though considerable effort has been devoted to planning and implementation of programs to mitigate health disparities in clinical trial recruitment, racial/ethnic minorities globally remain severely underrepresented in multiple myeloma clinical trials, which limits the generalizability of findings from cancer clinical research. In addition to novel recruitment strategies to ensure enrollment of underrepresented minorities, it is important to utilize existing real-world data to assess the geographic landscape of the racial/ethnic distribution within the patient population to aid in identification of target clinical trial recruitment goals to achieve equity in representation in future trials.