TY - JOUR
T1 - Representation of women in clinical trials supporting FDA-approval of contemporary cancer therapies
AU - Kalathoor, Sujay
AU - Ghazi, Sanam
AU - Otieno, Beryl
AU - Babcook, Melissa A.
AU - Chen, Sunnia
AU - Nidhi, Neha
AU - Bae, Junu
AU - Pierre-Charles, Jovan
AU - Breathett, Khadijah
AU - Mazimba, Sula
AU - Johnson, Amber
AU - Brewer, La Princess
AU - Mohammed, Selma
AU - Carter, Rebecca R.
AU - Bonsu, Janice M.
AU - Ferdousi, Mussammat
AU - Kola-Kehinde, Onaopepo
AU - McLaughlin, Eric
AU - Brammer, Jonathan
AU - Ruz, Patrick
AU - Khan, Sarah
AU - Odei, Bismarck
AU - Mitchell, Darrion
AU - Wei, Lai
AU - Patel, Prem
AU - Paskett, Electra D.
AU - Addison, Daniel
N1 - Publisher Copyright:
© 2024 The Author(s). International Journal of Cancer published by John Wiley & Sons Ltd on behalf of UICC.
PY - 2024/12/1
Y1 - 2024/12/1
N2 - Contemporary anticancer therapies frequently have different efficacy and side effects in men and women. Yet, whether women are well-represented in pivotal trials supporting contemporary anticancer drugs is unknown. Leveraging the Drugs@FDA database, clinicaltrials.gov, MEDLINE, and publicly available FDA-drug-reviews, we identified all pivotal (phase II and III) non-sex specific trials supporting FDA-approval of anticancer drugs (1998–2018). Observed-enrollment-rates were compared to expected-population-rates derived from concurrent US-National-Cancer-Institute's Surveillance-Epidemiology-and-End-Results (SEER) reported rates and US-Census databases. Primary outcome was the proportional representation of women across trials, evaluated by a participation-to-prevalence ratio (PPR), according to cancer type. Secondary outcome was the report of any sex-specific analysis of efficacy and/or safety, irrespective of treatment-arm. Overall, there were 148 trials, enrolling 60,216 participants (60.5 ± 4.0 years, 40.7% female, 79.1% biologic, targeted, or immune-based therapies) evaluating 99 drugs. Sex was reported in 146 (98.6%) trials, wherein 40.7% (24,538) were women, compared to 59.3% (35,678) men (p <.01). Altogether, women were under-represented in 66.9% trials compared to the proportional incidence of cancers by respective disease type; weight-average PPR of 0.91 (relative difference: -9.1%, p <.01). Women were most under-represented in gastric (PPR = 0.63), liver (PPR = 0.71), and lung (PPR =.81) cancer trials. Sex-based safety data was reported in 4.0% trials. There was no association between adequate female enrollment and drug efficacy (HR: 0.616 vs. 0.613, p =.96). Over time, there was no difference in the percentage of women recruited into clinical trials. Among pivotal clinical trials supporting contemporary FDA-approved cancer drugs, women were frequently under-represented and sex-specific-efficacy and safety-outcomes were commonly not reported.
AB - Contemporary anticancer therapies frequently have different efficacy and side effects in men and women. Yet, whether women are well-represented in pivotal trials supporting contemporary anticancer drugs is unknown. Leveraging the Drugs@FDA database, clinicaltrials.gov, MEDLINE, and publicly available FDA-drug-reviews, we identified all pivotal (phase II and III) non-sex specific trials supporting FDA-approval of anticancer drugs (1998–2018). Observed-enrollment-rates were compared to expected-population-rates derived from concurrent US-National-Cancer-Institute's Surveillance-Epidemiology-and-End-Results (SEER) reported rates and US-Census databases. Primary outcome was the proportional representation of women across trials, evaluated by a participation-to-prevalence ratio (PPR), according to cancer type. Secondary outcome was the report of any sex-specific analysis of efficacy and/or safety, irrespective of treatment-arm. Overall, there were 148 trials, enrolling 60,216 participants (60.5 ± 4.0 years, 40.7% female, 79.1% biologic, targeted, or immune-based therapies) evaluating 99 drugs. Sex was reported in 146 (98.6%) trials, wherein 40.7% (24,538) were women, compared to 59.3% (35,678) men (p <.01). Altogether, women were under-represented in 66.9% trials compared to the proportional incidence of cancers by respective disease type; weight-average PPR of 0.91 (relative difference: -9.1%, p <.01). Women were most under-represented in gastric (PPR = 0.63), liver (PPR = 0.71), and lung (PPR =.81) cancer trials. Sex-based safety data was reported in 4.0% trials. There was no association between adequate female enrollment and drug efficacy (HR: 0.616 vs. 0.613, p =.96). Over time, there was no difference in the percentage of women recruited into clinical trials. Among pivotal clinical trials supporting contemporary FDA-approved cancer drugs, women were frequently under-represented and sex-specific-efficacy and safety-outcomes were commonly not reported.
KW - FDA
KW - cancer therapies
KW - clinical trials
KW - gender
KW - safety analyses
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U2 - 10.1002/ijc.35110
DO - 10.1002/ijc.35110
M3 - Article
C2 - 39155749
AN - SCOPUS:85201380375
SN - 0020-7136
VL - 155
SP - 1958
EP - 1968
JO - International Journal of Cancer
JF - International Journal of Cancer
IS - 11
ER -