Assessment of gender representation in clinical trials leading to FDA approval for oncology therapeutics between 2014 and 2019: A systematic review‐based cohort study

Abstract

Background Ensuring representative data accrual in clinical trials is important to safeguard the generalizability of results and to minimize disparities in care. This study's goal was to evaluate differences in gender representation in trials leading to US Food and Drug Administration (FDA) cancer drug approvals. Methods An observational study was conducted from January 2014 to April 2019 using PubMed and the National Institutes of Health trials registry for primary trial reports. The National Cancer Institute's Surveillance, Epidemiology, and End Results program and US Census were consulted for national cancer incidence. The outcome was an enrollment incidence disparity (EID), which was calculated as the difference between male and female trial enrollment and national incidence, with positive values representing male overrepresentation. Results There were 149 clinical trials with 59,988 participants—60.3% and 39.7% were male and female, respectively—leading to 127 oncology drug approvals. The US incidence rates were 55.4% for men versus 44.6% for women. Gender representation varied by specific tumor type. Most notably, women were underrepresented in thyroid cancer (EID, +27.4%), whereas men were underrepresented in soft tissue cancer (EID, –26.1%). Overall, women were underrepresented when compared with expected incidence (EID, +4.9%; 42% of trials). Conclusions For many specific tumor types, women are underrepresented in clinical trials leading to FDA oncology drug approvals. It is critical to better align clinical trial cohort demographics and the populations to which these data will be extrapolated. Lay Summary This study assesses whether gender disparities exist in clinical trials leading to US Food and Drug Administration (FDA) cancer drug approvals. From January 2014 to April 2019, 149 clinical trials leading to FDA oncology drug approvals showed 60.3% and 39.7% of the enrollees were male and female, respectively. Gender representation varied by specific tumor when compared with the expected incidence rate of cancer in the United States, although women were more often underrepresented. Increased efforts are needed with regard to ensuring equitable representation in oncology clinical trials.

Enrollment of dengue patients in a prospective cohort study in Umphang District, Thailand, during the COVID‐19 pandemic: Implications for research and policy

“…21 Thailand as a whole also saw substantial declines in numbers of reported dengue cases from 2019 to 2020 and again to 2021. 29 Reasons included higher immunity following peak rates in Gender imbalance is persistent in many cohort studies, 30,31 including those conducted in urban areas. Two potential solutions to facilitate participation, where feasible, are to utilize home visits or telehealth technology.…”

“…Gender imbalance is persistent in many cohort studies, 30,31 including those conducted in urban areas. Two potential solutions to facilitate participation, where feasible, are to utilize home visits or telehealth technology.…”

Participant characteristics and exclusion from trials: a meta-analysis of individual participant-level data from phase 3/4 industry-funded trials in chronic medical conditions

“…Despite commitments from funders, journal editors, trialists and policymakers to improve the recruitment and retention of people from underserved populations 2 , there has not been any evident change over the last decade 1,4,5,[12][13][14] .…”

Dobbs v Jackson- Rewriting Women's Autonomy in Cancer Care

“…To improve understanding of biological differences in cancer outcomes and to ensure the safety and efficacy of oncology treatments, it is paramount that participants in clinical trials are representative of the real-world population. Women and minorities are underrepresented in clinical trials, [12][13][14][15] unknown effects of the drug on the fetus/newborn. With the overturning of Roe v Wade, women of childbearing age with a cancer diagnosis may feel discouraged and/or threatened by participating in clinical trials as therapeutic interventional studies would require documentation of regular pregnancy screening.…”