Certain groups remain underrepresented in pivotal clinical trials for leukemia and multiple myeloma (MM), a new study suggests.
The study showed that Black, Native American, and Hispanic patients were underrepresented across trials. Women were underrepresented in trials of acute myeloid leukemia (AML), and men were underrepresented in trials of MM and chronic myeloid leukemia (CML).
Researchers reported these findings in the Journal of Clinical Oncology.
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For this study, the researchers looked at clinical trials leading to drug approvals by the US Food and Drug Administration (FDA). The team collected demographic and geographic data from ClinicalTrials.gov and from primary manuscripts.
The study included 61 clinical trials leading to drug approvals between January 1, 2011, and October 1, 2021. A total of 41 trials reported data on race, 20 reported data on ethnicity, and 39 reported data on sex.
Together, the 41 trials with race data included 13,731 patients. Most patients (81.6%) were White, 9.1% were Asian/Pacific Islander, 3.8% were Black, 0.12% were American Indian or Alaska Native, and 1.5% were classified as “other” race. Across the trials with ethnicity data, 4.7% of patients were Hispanic.
Asian/Pacific Islander and Black patients had the highest representation in trials of CML (12.7% and 5.3%, respectively) and the lowest in trials of chronic lymphocytic leukemia (3% and 1.1%, respectively).
In general, the proportions of patients who were Black, Native American, or Hispanic were low, and these patients were underrepresented in the trials when compared with the general population.
“For example, although 27.4% of the US population diagnosed with MM identifies as Black, only 4.7% are represented in CTs [clinical trials] that led to FDA approval,” the researchers wrote.
Additionally, the researchers found that women were underrepresented in AML trials, with 44.7% of participants identifying as women and 60.5% identifying as men (P <.0001).
On the other hand, men were underrepresented in trials of MM (55.3% vs 60.2%; P <.0001) and CML (55.2% v 62.9%; P <.0001).
“[E]fforts to increase representation of minorities and disadvantaged populations in CTs [clinical trials] should be prioritized,” the researchers wrote. “Broader coverage by third-party payors (including Medicare and Medicaid) across borders, sponsor support to facilitate access of those with difficulties in accessing trial sites, and trial designs that are more patient-oriented allowing the participants to stay local should be enhanced to alleviate the disparities highlighted in our analysis.”
Disclosures: One study author declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.
Reference
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. Published online August 9, 2022. doi:10.1200/JCO.22.00504
