Participation of Women and Sex Analyses in Late-Phase Clinical Trials of New Molecular Entity Drugs and Biologics Approved by the FDA in 2007–2009

Poon, Rita; Khanijow, Keshav; Umarjee, S.; Fadiran, Emmanuel O.; Yu, Mengmeng; Zhang, Lei; Parekh, Ameeta

doi:10.1089/jwh.2012.3753

Cited by 69 publications

(77 citation statements)

References 9 publications

(13 reference statements)

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“…While the scientific community continues to combat male bias in pre-clinical research, representation of women in late-phase clinical trials has improved markedly in recent years 21 . The NIH initiative to increase the use of female organisms and cells in pre-clinical research ensures that the scientific community as a whole will grow to fully address the health of both men and women.…”

Section: Discussionmentioning

confidence: 99%

Breaking the Cycle

et al. 2016

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Despite the striking differences between male and female physiology, female physiology is understudied. In response, the National Institutes of Health is promulgating new policies to increase the use of female organisms in pre-clinical research. Females are commonly believed to have greater variability than males due to the estrous cycle, but recent studies call this belief into question. Effects of estrous cycling on mean arterial pressure were assessed in female Dahl S rats using telemetry and vaginal cytometry, and found that estrous cycling did not affect MAP magnitude or variance. Data from the PhysGen arm of the Program for Genomic Applications was used to compare male and female variance and coefficient of variation in 142 heart, lung, vascular, kidney and blood phenotypes, each measured in hundreds to thousands of individual rats from over 50 inbred strains. 74 of 142 phenotypes from this dataset demonstrated a sex difference in variance; however, 59% of these phenotypes exhibited greater variance in male rats rather than female. Remarkably, a retrospective power analysis demonstrated that only 16 of 74 differentially variable phenotypes would be detected when using an experimental cohort large enough to detect a difference in magnitude. No overall difference in coefficient of variation between male and female rats was detected when analyzing these 142 phenotypes. We conclude that variability of 142 traits in male and female rats is similar, suggesting that differential treatment of males and females for the purposes of experimental design is unnecessary until proven otherwise, rather than the other way around.

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Section: Discussionmentioning

confidence: 99%

Breaking the Cycle

et al. 2016

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“…6 In 2006-2009, about 40-60% of non-genderspecific trials enrolled female participants with safety and efficacy gender analyses in 74% of NDAs while 100% of Biologics License Applications (BLAs) had efficacy gender analysis. 7,8 There are many challenges in HIV vaccine research and development such as absence of known immune correlates of protection and lack of validated animal models. Clinical trials on promising vaccine candidates require target participants, especially young adults including women and adolescents.…”

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confidence: 99%

Issues in Women's Participation in a Phase III Community HIV Vaccine Trial in Thailand

Kaewkungwal

Pitisuttithum

Rerks-Ngarm

et al. 2013

AIDS Research and Human Retroviruses

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To assess qualities and outcomes of women participating in a large, community-based HIV vaccine trial, the present study was conducted among female participants of the RV 144 prime-boost trial in Thailand from 2003 to 2009. Qualities of participation refer to complete vaccination, retention, and status change. Outcomes of participation refer to incident rate, adverse event, and participation impact event. A total of 6,334 (38.6%) women participated in the trial, of whom about 50% were classified as low risk and 11% as high risk. About 85% of participants completed four vaccinations and 76% were included in the per-protocol analysis of the on-time vaccination schedule. More women (88%) completed 42 months follow-up compared with men (85%). Women aged 21 and above had more adverse events compared to younger age groups. More women (5%) compared with men (3%) reported participation impact events (PIEs). High-risk women had more PIEs and a higher infection rate compared to the low-risk group. Complete vaccination and retention on last follow-up were more common in married women aged above 21, and being a housewife. Female volunteers showed the same qualities and outcomes of participation as males in the HIV vaccine trial. There was no statistically significant difference in vaccine efficacy between men and women, especially among the high-risk and married women. The study highlighted the important behavioral, social, and cultural issues that could be considered for future HIV vaccine trial designs.

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“…We also planned to calculate a participation to prevalence ratio (PPR), which can be used to consider the representation of a group of interest (e.g., women) in the trial relative to their proportion in the overall disease population (Poon et al, 2013;Scott et al, 2018). We calculated the PPR by dividing the proportion of consented women among total trial participants by the proportion of women in the overall population of individuals with the disease or condition in question.…”

Section: Analytic Planmentioning

confidence: 99%

Enrollment and Retention of Men and Women in Health Services Research and Development Trials

Goldstein

Duan-Porter

Alkon

et al. 2019

Women's Health Issues

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Introduction: Sex-and gender-specific science is essential to inform patient-centered, evidence-based care. Developing such evidence requires adequate inclusion of both women and men in trials. We sought to describe study participation of women and men in Department of Veterans Affairs Health Services Research and Development trials. Methods: We identified recent health services research trials from one Health Services Research and Development Center of Innovation and compared the participation of women and men from trial recruitment to study completion. We also calculated the participation to prevalence ratio (PPR) by sex for each trial. Results: We included eight trials that started recruitment between 2011 and 2014. Only one study purposefully attempted to boost the recruitment of women. Overall, the PPR for women ranged from 0.2 to 4.5, with seven studies having a PPR of greater than 1, indicating that women participated in these trials at proportions greater than their prevalence in the disease population within the Department of Veterans Affairs. The PPR for men ranged from 0.8 to 1.1. Retention was best with those studies that used administrative data for final outcomes assessment. No studies provided results stratified by sex or conducted analyses to explore treatment effect by sex. Conclusions: At a single site, women participated in Health Services Research and Development trials at similar or greater rates to men without cross-study efforts to enrich the recruitment or retention of women. Adding strategic recruitment approaches could further boost the proportion of women in Department of Veterans Affairs trials and enable adequately powered sex-based analyses. Published by Elsevier Inc. on behalf of Jacobs Institute of Women's Health. Sex-and gender-based differences in the pathophysiology, quality of care, and patient experiences of disease require sexand gender-specific science to inform the provision of patientcentered, evidence-based care. For example, when compared with men, women with coronary artery disease are less likely to take statins and antiplatelet medications (Johansen, Hefner, & Foraker, 2015), and respond differently to pharmacologic interventions for pain (Bartley & Fillingim, 2013). Acquiring the evidence needed to optimize patient-centered care for women Supported by the Durham Center of Innovation to Accelerate Discovery and Practice Transformation (ADAPT) (CIN 13-410

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Participation of Women and Sex Analyses in Late-Phase Clinical Trials of New Molecular Entity Drugs and Biologics Approved by the FDA in 2007–2009

Cited by 69 publications

References 9 publications

Breaking the Cycle

Breaking the Cycle

Issues in Women's Participation in a Phase III Community HIV Vaccine Trial in Thailand

Enrollment and Retention of Men and Women in Health Services Research and Development Trials

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