Race and Ethnicity Reporting and Representation in Pediatric Clinical Trials

Abstract

BACKGROUND AND OBJECTIVES Representative enrollment of racial and ethnic minoritized populations in biomedical research ensures the generalizability of results and equitable access to novel therapies. Previous studies on pediatric clinical trial diversity are limited to subsets of journals or disciplines. We aimed to evaluate race and ethnicity reporting and representation in all US pediatric clinical trials on ClinicalTrials.gov. METHODS We performed a cross-sectional study of US-based clinical trials registered on ClinicalTrials.gov that enrolled participants aged <18 years old between October 2007 and March 2020. We used descriptive statistics, compound annual growth rates, and multivariable logistic regression for data analysis. Estimates of US population statistics and disease burden were calculated with the US Census, Kids’ Inpatient Database, and National Survey of Children’s Health. RESULTS Among 1183 trials encompassing 405 376 participants, race and ethnicity reporting significantly increased from 27% in 2007 to 87% in 2018 (P < .001). The median proportional enrollment of Asian American children was 0.6% (interquartile range [IQR], 0%–3.7%); American Indian, 0% (IQR, 0%–0%); Black, 12% (IQR, 2.9%–28.4%); Hispanic, 7.1% (IQR, 0%–18.6%); and white 66.4% (IQR, 41.5%–81.6%). Asian American, Black, and Hispanic participants were underrepresented relative to US population demographics. Compared with expected proportions based on disease prevalence and hospitalizations, Asian American and Hispanic participants were most consistently underrepresented across diagnoses. CONCLUSIONS While race and ethnicity reporting in pediatric clinical trials has improved, the representative enrollment of minoritized participants remains an ongoing challenge. Evidence-based and policy solutions are needed to address these disparities to advance biomedical innovation for all children.

Behavior Problems in Low-Income Young Children Screened in Pediatric Primary Care

“…It is also possible that Black or African American caregivers are reluctant to report concerns about their child’s behavior for fear of the response of health care professionals (eg, may result in reports to child protective services as there is disproportionately higher contact among Black or African American families compared with White families due to structural racism) . Our findings underscore the need for increased cultural sensitivity and understanding of Black or African American families and other historically marginalized populations in pediatric primary care both in terms of clinical practice and research …”

“…20 Our findings underscore the need for increased cultural sensitivity and understanding of Black or African American families and other historically marginalized populations in pediatric primary care both in terms of clinical practice and research. 21,22 Being male was associated with the 3 elevated trajectories compared with the LS trajectory group. Males were also more represented in the HI vs MD groups although being female was associated with membership in the MD vs LI groups.…”

Reporting and Representation of Participant Race and Ethnicity in National Institutes of Health–Funded Pediatric Clinical Trials

“…The diversity of children included in clinical research has been examined in prior studies with varying results regarding representation of different racial and ethnic groups . One study analyzing data from 33 federally funded studies evaluating pediatric drugs and devices and enrolling almost 11 000 participants from 2008 to 2020 found no evidence of racial or ethnic bias in enrollment .…”

“…Despite these activities, a 2022 report from the National Academies of Sciences, Engineering, and Medicine on the representation of women and underrepresented groups in clinical research concluded that racial and ethnic diversity in clinical trials remained inadequate and largely unchanged over the past 30 years . Although the report did not specifically examine pediatric trial participants, several studies have assessed racial and ethnic diversity in pediatric studies across a spectrum of trial cohorts and presented mixed findings on the level of representation of different patient groups . Given the unique considerations in enrolling children into clinical trials, a focused assessment of the diversity of pediatric participants in NIH-funded clinical trials, including reporting practices for race and ethnicity information, could inform the impact of current policies and identify any remaining gaps in the NIH’s strategies.…”

Association of High-Dose Erythropoietin With Circulating Biomarkers and Neurodevelopmental Outcomes Among Neonates With Hypoxic Ischemic Encephalopathy

“…The trial was approved by the institutional review boards at all participating sites and was registered with the US Food and Drug Administration (Investigational New Drug application 102 138). Maternal race and ethnicity was self-reported and was assessed in this study because such reporting is important to determine whether there were differences in severity of HIE or in outcomes based on race, and whether the results are generalizable …”