Racial Disparities Seen in US Food and Drug Administration Drug Approval Trials in Multiple Myeloma

According to the results of a study published in Blood Advances, Black and Hispanic patients appear to be underrepresented in global clinical trials supporting US Food and Drug Administration (FDA) approval of multiple myeloma (MM) drugs.

Researchers conducted a pooled analysis of all pivotal clinical trials submitted to the FDA to support approval of an MM therapeutic between 2006 and 2019. The study aimed to characterize the demographic representation in MM clinical trials and evaluate outcomes based on race and ethnicity.

The researchers collected baseline demographic characteristics (such as age, sex, race, ethnicity, and country) and baseline patient and disease characteristics (including prior autologous stem cell transplantation, prior lines of therapy, and Eastern Cooperative Oncology Group performance status). They pooled the data for the analyses and used an age-adjusted stratified Cox regression model to evaluate the relationship between time-to-event outcomes and race and ethnicity.

The team identified 19 global trials, 4 single arm trials and 15 randomized controlled trials, comprising 10 157 patients. The pooled patient population was 84% White, 7% Asian, 4% Black, 4% Hispanic, 0.10% Native Hawaiian or other Pacific Islander, and 0.04% American Indian or Alaska Native. The median patient age was 68 years (interquartile range, 60-73).

Approximately 17% of patients were enrolled in trials in the US, while 85% were enrolled in trials throughout the rest of the world (RoW). The researchers found that the proportion of Black and Hispanic patients enrolled in US trials was higher than that of trials in the RoW (18% vs 1% and 6% vs 4%, respectively); the proportion of Asian patients enrolled in US trials was lower than that of trials in the RoW (1% vs 8%).

The researchers observed an age-adjusted overall survival hazard ratio (HR) of 0.89 (95% confidence interval [CI], 0.75-1.05) for Black patients compared with White patients. For US trials, the age-adjusted HR for Black patients compared with White patients was 0.82 (95% CI, 0.66-1.02). For RoW trials, the age-adjusted HR for Black patients compared with White patients was 1.31 (95% CI, 0.97-1.77).

“Black and Hispanic patients were underrepresented in the trials supporting FDA approval of MM drugs,” concluded the researchers. “Given the higher incidence of MM in [Black patients] and the different disease characteristics, efforts should be made to improve representation of [Black patients] in MM clinical trials.”

Limitations of the study included a lack of adjustment in the analysis for multiplicity, inability to assess the potential impact of treatment on race- and ethnicity-based outcomes due to small sample sizes, treatment heterogeneity, and combination regimens, and lack of generalizability.

Disclosure: Some study authors declared affiliations with the US Food and Drug Administration, a federal entity. Please see the original reference for a full list of authors’ disclosures. 

Reference

Kanapuru B, Fernandes LL, Fashoyin-Aje LA, et al. Analysis of racial and ethnic disparities in multiple myeloma US FDA drug approval trials. Blood Adv. 2022;6(6):1684-1691. doi:10.1182/bloodadvances.2021005482

This article originally appeared on Hematology Advisor