Sujay Kalathoor, Sanam Ghazi, Beryl Otieno, Melissa A Babcook, Sunnia Chen, Neha Nidhi, Junu Bae, Jovan Pierre-Charles, Khadijah Breathett, Sula Mazimba, Amber Johnson, LaPrincess Brewer, Selma Mohammed, Rebecca R Carter, Janice M Bonsu, Mussammat Ferdousi, Onaopepo Kola-Kehinde, Eric McLaughlin, Jonathan Brammer, Patrick Ruz, Sarah Khan, Bismarck Odei, Darrion Mitchell, Lai Wei, Prem Patel, Electra D Paskett, Daniel Addison
{"title":"妇女在支持 FDA 批准当代癌症疗法的临床试验中的代表性。","authors":"Sujay Kalathoor, Sanam Ghazi, Beryl Otieno, Melissa A Babcook, Sunnia Chen, Neha Nidhi, Junu Bae, Jovan Pierre-Charles, Khadijah Breathett, Sula Mazimba, Amber Johnson, LaPrincess Brewer, Selma Mohammed, Rebecca R Carter, Janice M Bonsu, Mussammat Ferdousi, Onaopepo Kola-Kehinde, Eric McLaughlin, Jonathan Brammer, Patrick Ruz, Sarah Khan, Bismarck Odei, Darrion Mitchell, Lai Wei, Prem Patel, Electra D Paskett, Daniel Addison","doi":"10.1002/ijc.35110","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":180,"journal":{"name":"International Journal of Cancer","volume":null,"pages":null},"PeriodicalIF":5.7000,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Representation of women in clinical trials supporting FDA-approval of contemporary cancer therapies.\",\"authors\":\"Sujay Kalathoor, Sanam Ghazi, Beryl Otieno, Melissa A Babcook, Sunnia Chen, Neha Nidhi, Junu Bae, Jovan Pierre-Charles, Khadijah Breathett, Sula Mazimba, Amber Johnson, LaPrincess Brewer, Selma Mohammed, Rebecca R Carter, Janice M Bonsu, Mussammat Ferdousi, Onaopepo Kola-Kehinde, Eric McLaughlin, Jonathan Brammer, Patrick Ruz, Sarah Khan, Bismarck Odei, Darrion Mitchell, Lai Wei, Prem Patel, Electra D Paskett, Daniel Addison\",\"doi\":\"10.1002/ijc.35110\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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. 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引用次数: 0
摘要
当代抗癌疗法对男性和女性的疗效和副作用往往不同。然而,在支持当代抗癌药物的关键性试验中,女性是否具有充分的代表性还不得而知。利用 Drugs@FDA 数据库、clinicaltrials.gov、MEDLINE 和公开的 FDA 药物评论,我们确定了所有支持 FDA 批准抗癌药物的关键性(II 期和 III 期)非性别特定试验(1998-2018 年)。我们将观察到的注册率与同时从美国国家癌症研究所的 "监测-流行病学-最终结果"(SEER)报告率和美国人口普查数据库中得出的预期人口比例进行了比较。主要结果是根据癌症类型,通过参与率与患病率之比(PPR)评估女性在各项试验中的比例。次要结果是对疗效和/或安全性的性别特异性分析报告,与治疗臂无关。总体而言,共有 148 项试验,60216 名参与者(60.5 ± 4.0 岁,40.7% 为女性,79.1% 为生物、靶向或免疫疗法)参与了 99 种药物的评估。146项(98.6%)试验报告了性别,其中女性占40.7%(24,538人),男性占59.3%(35,678人)(p
Representation of women in clinical trials supporting FDA-approval of contemporary cancer therapies.
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.
期刊介绍:
The International Journal of Cancer (IJC) is the official journal of the Union for International Cancer Control—UICC; it appears twice a month. IJC invites submission of manuscripts under a broad scope of topics relevant to experimental and clinical cancer research and publishes original Research Articles and Short Reports under the following categories:
-Cancer Epidemiology-
Cancer Genetics and Epigenetics-
Infectious Causes of Cancer-
Innovative Tools and Methods-
Molecular Cancer Biology-
Tumor Immunology and Microenvironment-
Tumor Markers and Signatures-
Cancer Therapy and Prevention