现代抗癌疗法通常对男性和女性具有不同的功效和副作用。然而，女性在支持当代抗癌药物的关键试验中是否有充分代表性尚不清楚。利用 Drugs@FDA 数据库、clinicaltrials.gov、MEDLINE 和公开的 FDA 药物评论，我们确定了支持 FDA 批准抗癌药物的所有关键（II 期和 III 期）非性别特异性试验（1998-2018 年）。将观察到的入学率与来自美国国家癌症研究所的监测流行病学和最终结果 (SEER) 报告率和美国人口普查数据库的预期人口率进行比较。主要结果是试验中女性的比例，根据癌症类型通过参与患病率（PPR）进行评估。次要结果是任何性别特异性疗效和/或安全性分析的报告，无论治疗组如何。总体而言，共有 148 项试验，招募了 60,216 名参与者（60.5±4.0 岁，40.7% 女性，79.1% 生物、靶向或免疫疗法）评估 99 种药物。 146 项试验 (98.6%) 报告了性别，其中 40.7% (24,538) 为女性，而男性为 59.3% (35,678) (p<0.01)。总体而言，与各疾病类型的癌症发病率比例相比，女性在 66.9% 的试验中代表性不足；重均 PPR 为 0.91（相对差异：-9.1%，p < .01）。女性在胃癌 (PPR = 0.63)、肝癌 (PPR = 0.71) 和肺癌 (PPR = .81) 癌症试验中的代表性最低。 4.0% 的试验报告了基于性别的安全数据。足够的女性入组率与药物疗效之间不存在关联（HR：0.616 vs. 0.613，p = .96）。随着时间的推移，招募参加临床试验的女性比例没有变化。在支持当代 FDA 批准的癌症药物的关键临床试验中，女性的代表性常常不足，并且通常没有报告特定性别的疗效和安全性结果。© 2024 作者。约翰·威利出版的《国际癌症杂志》

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.© 2024 The Author(s). International Journal of Cancer published by John Wiley & Sons Ltd on behalf of UICC.