Representation of women in clinical trials supporting FDA-approval of contemporary cancer therapies

被引：0

作者：

Kalathoor, Sujay ^{[1
]}

Ghazi, Sanam ^{[1
]}

Otieno, Beryl ^{[1
,2
]}

Babcook, Melissa A. ^{[1
,3
,4
]}

Chen, Sunnia ^{[1
]}

Nidhi, Neha ^{[1
]}

Bae, Junu ^{[1
]}

Pierre-Charles, Jovan ^{[1
]}

Breathett, Khadijah ^{[5
]}

Mazimba, Sula ^{[6
]}

Johnson, Amber ^{[7
]}

Brewer, LaPrincess ^{[8
]}

Mohammed, Selma ^{[9
]}

Carter, Rebecca R. ^{[1
,10
]}

Bonsu, Janice M. ^{[1
]}

Ferdousi, Mussammat ^{[1
]}

Kola-Kehinde, Onaopepo ^{[1
]}

McLaughlin, Eric ^{[11
]}

Brammer, Jonathan ^{[4
,12
]}

Ruz, Patrick ^{[1
]}

Khan, Sarah ^{[1
]}

Odei, Bismarck ^{[4
,13
]}

Mitchell, Darrion ^{[13
]}

Wei, Lai ^{[11
]}

Patel, Prem ^{[1
]}

Paskett, Electra D. ^{[14
]}

Addison, Daniel ^{[1
,14
]}

机构：

[1] Ohio State Univ, Med Ctr, Div Cardiol, Cardiooncol Program, Columbus, OH USA

[2] Greater Baltimore Med Ctr, Dept Med, Baltimore, MD USA

[3] Ohio State Univ, James Canc Hosp, Div Oncol, Columbus, OH USA

[4] Ohio State Univ, Solove Res Inst, Columbus, OH USA

[5] Indiana Univ, Div Cardiol, Indianapolis, IN USA

[6] Univ Virginia, Div Cardiol, Charlottesville, VA USA

[7] Univ Pittsburgh, Div Cardiol, Sch Med, Pittsburgh, PA USA

[8] Mayo Clin, Div Cardiol, Rochester, MN USA

[9] Creighton Univ, Div Cardiol, Omaha, NE USA

[10] Ohio State Univ, Coll Med, Ctr Adv Team Sci Analyt & Syst Thinking CATALYST, Columbus, OH USA

[11] Ohio State Univ, Dept Biomed Informat, Ctr Biostat, Columbus, OH USA

[12] Ohio State Univ, Div Hematol, James Canc Hosp, Columbus, OH USA

[13] Ohio State Univ, Dept Radiat Oncol, James Canc Hosp, Columbus, OH USA

[14] Ohio State Univ, Dept Internal Med, Div Canc Prevent & Control, Columbus, OH USA

来源：

INTERNATIONAL JOURNAL OF CANCER | 2024年 / 155卷 / 11期

基金：

美国国家卫生研究院;

关键词：

cancer therapies; clinical trials; FDA; gender; safety analyses; PARTICIPATION; SEX;

D O I：

10.1002/ijc.35110

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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, , 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.

引用

页码：1958 / 1968

页数：11

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