ORIGINAL RESEARCH article
Front. Oncol.
Sec. Cancer Epidemiology and Prevention
This article is part of the Research TopicEquity in Cancer Prevention and Early DetectionView all 11 articles
Equity and Representation in Oncology Clinical Evidence for FDA-Approved Treatments
Provisionally accepted
- China Pharmaceutical University, Nanjing, China
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Abstract Background Pivotal clinical trials form the evidentiary foundation for FDA oncology drug approvals, yet persistent disparities in participant demographics may limit the generalizability and equity of cancer care. This study aimed to quantify demographic disparities in pivotal trials that supported FDA oncology drug approvals, and to identify trial-level characteristics associated with the underrepresentation of women, racial and ethnic minorities, and older adults. Methods We systematically identified pivotal trials supporting FDA oncology drug approvals from January 2018 through November 2024 using the FDA Hematology/Oncology Approvals database. Demographic data were extracted for gender, race, ethnicity, and age. SEER 21 data were used to derive population-based estimates. Enrollment incidence ratios (EIRs) were calculated for gender, race, ethnicity, and older adults (EIR <1 indicating underrepresentation). Meta-regression was used to evaluate temporal trends and associations between trial characteristics and representation disparities. Subgroup analyses were conducted for pediatric trials, accelerated approvals, and other trial-level factors. Results Among 329 included trials enrolling over 164,000 participants, women, older adults, Black, and Hispanic individuals were consistently underrepresented. Pooled EIRs indicated significant underrepresentation of female (EIR, 0.83; 95% CI, 0.78–0.88), Black (EIR, 0.26; 95% CI, 0.22–0.30), Hispanic (EIR, 0.50; 95% CI, 0.44–0.57), and older participants (EIR, 0.86; 95% CI, 0.83–0.89). Meta-regression showed modest gains in female representation over time (β=0.02, p=0.03), but no significant improvement for Black or Hispanic participants. Older adult representation declined slightly. Trial-level factors associated with underrepresentation included industry sponsorship, use of overall survival as the primary endpoint, and accelerated approval designation. Conclusions Despite recent regulatory efforts, demographic disparities persist in pivotal oncology trials supporting FDA approvals. Female, Black, Hispanic, and older adults remain underrepresented, limiting the applicability of trial findings to real-world cancer populations. Greater efforts are needed to design inclusive trials, enforce diversity mandates, and ensure equitable evidence generation in oncology drug development.
Keywords: FDA approvals, Gender and age diversity, health equity, oncology clinical trials, racial and ethnic disparities
Received: 15 Jul 2025; Accepted: 30 Jan 2026.
Copyright: © 2026 Mao, Li and Cheng. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Ningying Mao
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