Diversity, Equity, and Inclusion in Gene and Cell Therapies Clinical Trials
Author(s)
Gerald O. Ozota, PhD1, Rudy Chang, PhD2, Enrique Seoane-Vazquez, PhD3, Lawrence Brown, PhD3;
1Chapman University School of Pharmacy, Pharmacoeconomics and Policy, Irvine, CA, USA, 2Chapaman University School of Pharmacy, Department of Biomedical and Pharmaceutical Sciences, Irvine, CA, USA, 3Chapaman University School of Pharmacy, Pharmacoeconomics and Policy, Irvine, CA, USA
1Chapman University School of Pharmacy, Pharmacoeconomics and Policy, Irvine, CA, USA, 2Chapaman University School of Pharmacy, Department of Biomedical and Pharmaceutical Sciences, Irvine, CA, USA, 3Chapaman University School of Pharmacy, Pharmacoeconomics and Policy, Irvine, CA, USA
OBJECTIVES: Advanced therapy medicinal products (ATMP), including gene and cell therapies, are rapidly evolving, offering alternatives for unmet medical needs. ATMP exhibit inter-individual variability affecting pharmacodynamics, pharmacokinetics, safety, and efficacy, highlighting the importance of diversity, equity, and inclusion (DEI) in clinical trials. This study evaluated DEI in clinical trials for gene and cell therapy authorized by the FDA as of October/ 31, 2024.
METHODS: We collected FDA regulatory information for ATMP authorized from 2000 through October 2024 from the FDA website. We conducted a descriptive analysis and a random-effects meta-analysis of underrepresentation and overrepresentation ratios for individual trials relative to the U.S.-based proportions of racial and ethnic groups for the specific disease states.
RESULTS: The FDA authorized 23 gene therapies, including 6(20.0%) CAR T-cell therapies, and 9 cell therapies as of October 31, 2024. There were 11(36.7%) ATMP indicated for cancer. The FDA reviewed 32 clinical trials for those ATMP with a total of 3,519±111.9 participants. Of these trials, 27(84.4%) had more than 30 participants. The lowest enrollment was for atidarsagene autotemcel (7 participants), while sipuleucel-T had the highest (512 participants). Among the participants, 2,900(82.4%) identified as White, 233(6.62%) as Black, and 162(4.6%) as Asian. White participants were disproportionately represented, particularly in sipuleucel-T (90.0%) and talimogene laherparepvec (97.9%). Clinical trials including at least 30 minority participants occurred for Black individuals in only 2 cases (6.3%), for Asian individuals in 0 cases (0.0%), and for Hispanic individuals in 1 case (3.1%).
CONCLUSIONS: The findings revealed persistent racial disparities in gene and cell therapy clinical trials, with minority populations underrepresented and White participants comprising the majority. Achieving DEI in clinical trials remains a challenge, underscoring the need for greater efforts to address these disparities.
METHODS: We collected FDA regulatory information for ATMP authorized from 2000 through October 2024 from the FDA website. We conducted a descriptive analysis and a random-effects meta-analysis of underrepresentation and overrepresentation ratios for individual trials relative to the U.S.-based proportions of racial and ethnic groups for the specific disease states.
RESULTS: The FDA authorized 23 gene therapies, including 6(20.0%) CAR T-cell therapies, and 9 cell therapies as of October 31, 2024. There were 11(36.7%) ATMP indicated for cancer. The FDA reviewed 32 clinical trials for those ATMP with a total of 3,519±111.9 participants. Of these trials, 27(84.4%) had more than 30 participants. The lowest enrollment was for atidarsagene autotemcel (7 participants), while sipuleucel-T had the highest (512 participants). Among the participants, 2,900(82.4%) identified as White, 233(6.62%) as Black, and 162(4.6%) as Asian. White participants were disproportionately represented, particularly in sipuleucel-T (90.0%) and talimogene laherparepvec (97.9%). Clinical trials including at least 30 minority participants occurred for Black individuals in only 2 cases (6.3%), for Asian individuals in 0 cases (0.0%), and for Hispanic individuals in 1 case (3.1%).
CONCLUSIONS: The findings revealed persistent racial disparities in gene and cell therapy clinical trials, with minority populations underrepresented and White participants comprising the majority. Achieving DEI in clinical trials remains a challenge, underscoring the need for greater efforts to address these disparities.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR11
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling, Health Disparities & Equity
Disease
STA: Genetic, Regenerative & Curative Therapies