Analysis of racial and ethnic disparities in multiple myeloma US FDA drug approval trials

Area of Science:

• Hematology
• Clinical Trials
• Health Disparities

Background:

• Multiple myeloma (MM) disproportionately affects African Americans (AAs), with higher incidence and mortality rates compared to White patients.
• AAs also exhibit distinct genetic profiles, including a higher prevalence of immunoglobulin H translocations t(11;14) and t(14;16).

Purpose of the Study:

• To analyze the demographic representation of racial and ethnic groups in multiple myeloma clinical trials.
• To evaluate treatment outcomes based on race and ethnicity in these trials.

Main Methods:

• A pooled analysis of 19 global clinical trials supporting US Food and Drug Administration (FDA) approval for MM therapeutics (2006-2019) was conducted.
• Demographic data were analyzed, and age-adjusted Cox regression models assessed time-to-event outcomes stratified by race and ethnicity.

Main Results:

• White patients constituted 84% of the 10,157 participants; Black patients represented 4% and Hispanic patients 4%.
• Black patients were predominantly enrolled in US-based trials. Overall survival showed a trend favoring Black patients in the US (HR 0.82) but was less favorable outside the US (HR 1.31).
• Significant underrepresentation of Black and Hispanic populations was observed in trials supporting MM drug approvals.

Conclusions:

• African Americans and Hispanic patients remain underrepresented in pivotal multiple myeloma clinical trials.
• The geographic distribution of Black patients in trials (primarily US-based) may influence outcome interpretations.
• Increased efforts are crucial to enhance the inclusion of diverse populations, particularly African Americans, in MM clinical trials to ensure generalizability of findings and equitable therapeutic development.