To measure demographic inequalities in pivotal trials supporting FDA oncology drug approvals and identify specific trial-level attributes linked to the underrepresentation of women, racial and ethnic minorities, and older individuals, as well as evaluate the impact of COVID-19 on participant representation.
Key Findings:
Women (EIR 0.83, 95% CI: 0.78–0.88), Black individuals (EIR 0.26, 95% CI: 0.22–0.30), Hispanic individuals (EIR 0.50, 95% CI: 0.44–0.57), and older adults (EIR 0.86, 95% CI: 0.83–0.89) were underrepresented in trials.
Slight improvement in female representation over time (β=0.02, p=0.03), but no significant changes for Black or Hispanic individuals.
Older adult representation showed a minor decrease.
Trial-level factors linked to underrepresentation included industry sponsorship and use of overall survival as the primary endpoint.
EIRs during the pandemic were comparable to non-pandemic times, with diminished representation for older adults and White individuals.
Interpretation:
Demographic inequalities persist in oncology trials, limiting the generalizability of trial outcomes to actual cancer demographics, which may adversely affect treatment efficacy across diverse populations.
Limitations:
Potential biases in trial selection and demographic reporting that may skew results.
Exclusion of device-related approvals and studies lacking complete demographic data.
Conclusion:
Increased efforts are necessary to create inclusive trials, implement diversity requirements, and ensure fair evidence creation in oncology drug development.
