Enrollment of Black Participants in Pivotal Clinical Trials Supporting US Food and Drug Administration Approval of Chimeric Antigen Receptor–T Cell Therapy for Hematological Malignant Neoplasms

Key Points

Question

What is the proportion of Black participants enrolled in pivotal clinical trials supporting US Food and Drug Administration approval of chimeric antigen receptor–T cell (CAR-T) therapy for hematological malignant neoplasms?

Findings

In this cross-sectional study of data from 7 clinical trials that resulted in a subsequent Food and Drug Administration CAR-T therapy approval for hematological malignant neoplasms, the proportion of Black participants, adjusted to the disease prevalence, was suboptimal.

Meaning

The findings of this study suggest that disparities in the use of CAR-T therapy in clinical trials for hematological malignant neoplasms exist and are disproportionately affecting Black participants.

Abstract

This cross-sectional study examines the enrollment of Black participants in clinical trials that resulted in a subsequent US Food and Drug Administration (FDA) approval of chimeric antigen receptor–T cell therapy products for hematological malignant neoplasms.

Abstract

Importance

Disparities that affect Black persons with various hematological malignant neoplasms are substantial, yet little is known about disparities related to the use of US Food and Drug Administration (FDA)–approved chimeric antigen receptor–T cell (CAR-T) therapy.

Objective

To examine the enrollment of Black participants in clinical trials that resulted in a subsequent FDA approval of CAR-T products in hematological malignant neoplasms.

Design, Setting, and Participants

A cross-sectional study was performed using publicly available data on drug products and demographic subgroups from Drugs@fda in the period of August 2017 to May 2021. Data analysis included patients with large B cell lymphoma, follicular lymphoma, mantle cell lymphoma, acute lymphoblastic leukemia, and multiple myeloma who were enrolled into 7 clinical trials that investigated various CAR-T products. The study was conducted from July 1, 2021, to November 30, 2021.

Main Outcomes and Measures

Frequencies of participation of Black participants were calculated with adjustment for disease prevalence.

Results

Of the 1057 enrolled patients included in the study, CAR-T products were given to 746 patients (71%), and efficacy was reported for 729 enrolled patients (69%) across all the approved CAR-T products and indications. Most patients (1015 patients [96%]) were enrolled in the US. Black participants were included in the racial category other in the study that supported tisagenlecleucel approval in acute lymphoblastic leukemia; otherwise, their enrollment was specified either in the study publication and/or the demographic subgroup information available under the FDA product labeling information. The number of Black participants who received the CAR-T product and had reported efficacy varied between studies (range, 1-12 participants [2%-5%]). Adjusted prevalence measures showed the lowest participation to prevalence ratio of 0.2 for multiple myeloma and 0.6 for large B cell lymphoma.

Conclusions and Relevance

The findings of this study suggest that there are substantial disparities affecting Black patients across all approved CAR-T products used to treat hematological malignant neoplasms with otherwise limited effective treatment options. The study findings might aid policy discussions regarding the immediate need of regulations that enforce certain thresholds of Black patients’ enrollment before granting FDA approval.