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Regulatory Focus™ > News Articles > 2022 > 4 > Study: Black patients underrepresented in pivotal CAR T-cell trials

Study: Black patients underrepresented in pivotal CAR T-cell trials

Posted 20 April 2022 | By Mary Ellen Schneider 

Study: Black patients underrepresented in pivotal CAR T-cell trials

The number of Black participants who received chimeric antigen receptor (CAR) T-cell therapies for hematologic cancers in pivotal clinical trials ranged from about 2% to 5%, suggesting the need for policy changes to increase trial enrollment of minority patients, according to an analysis published in JAMA Network Open.
“The findings of this study suggest that low enrollment of Black persons exists in trials for CAR-T therapy and that the disparity is substantial and ongoing, especially for therapies to treat [multiple myeloma],” wrote Samer Al Hadidi, of the University of Arkansas for Medical Sciences, and colleagues. “Efforts should be made to understand and overcome barriers that lead to decreased enrollment of Black participants in clinical trials that include novel, potentially beneficial, and/or curative CAR-T therapies in difficult-to-treat hematological malignant neoplasms with otherwise limited treatment options.”
The cross-sectional study examined demographic data across seven clinical trials that resulted in approval of a CAR T-cell therapies by the US Food and Drug Administration (FDA) between August 2017 and May 2021. The researchers examined the number of Black participants in each trial and calculated the participation to prevalence ratio (PPR). The PPR is based on the percentage of Black participants in the trial divided by the percentage of Black individuals in the disease population, with a range between 0.8 and 1.2 reflecting similar representation in the trial and disease population.
Overall, the number of Black participants who received CAR T-cell products and had efficacy data reported varied between with the seven studies, with a range of 1-12 participants (about 2%-5%). When adjusted for disease prevalence, multiple myeloma studies had the lowest PPR of 0.2, while follicular lymphoma trials had the highest PPR of 0.8. The PPR for mantle cell lymphoma and large B-cell lymphoma was 0.4 and 0.6, respectively. The researchers could not calculate a PPR for acute lymphoblastic leukemia trials because of a lack of data on enrolled Black participants.
“The incidence of [multiple myeloma] is more than 2 times higher among Black persons than among non-Hispanic White persons,” the researchers wrote. “Black patients with [multiple myeloma] face multiple disparities, including lower use of hematopoietic stem cell transplantation, palliative care, and novel therapeutics, which may result in worse outcomes. Lack of access to novel therapies that disproportionately affect Black persons may result in further widening of the existing established disparities.”
The researchers suggested some potential policy efforts to encourage increased enrollment of Black participants, including allowing more medical centers that serve a higher percentage of Black patients to be part of ongoing CAR-T clinical trials. Another idea, the researchers explained, would be to implement the “least acceptable race-specific enrollment percentage” in registration trials over a period of 10 years. This percentage would be calculated based on the race-specific incidence of the studied cancer and the sample size needed for the trial to answer a clinical question.
The FDA has recently made a push to increase clinical trial diversity. In draft guidance issued on 13 April 2022, the agency recommended that sponsors include a “Race and Ethnicity Diversity Plan” when submitting an investigational new drug (IND), biologics license application (BLA) or investigational device exemption (IDE) application (RELATED: FDA recommends sponsors plan to include race, ethnicity in clinical trial design, Regulatory Focus 13 April 2022).
In November 2020, FDA issued final guidance for trial sponsors that calls for expanding enrollment criteria and minimizing patient exclusions (RELATED: FDA lays out strategies for promoting diversity in clinical trial enrollment, Regulatory Focus 09 November 2020).



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