Race and Ethnicity in Published Pediatric Clinical Trial Enrollment in the United States, 2011-2020

Background: Equitable representation of participants who are Black, Indigenous, and People of Color in clinical trials enhances inclusivity in the scientific process and generalizability of results.

Methods: We conducted a cross-sectional study of published articles reporting pediatric clinical trials conducted in the U.S. and published in five leading general pediatric and five leading general medical journals from January 1, 2011-December 31, 2020. We determined the reporting of participant race and ethnicity in published clinical trial results. We compared the number of children enrolled versus U.S. Census populations of pediatric racial and ethnic groups. We calculated the mean diversity index of all trials by year using linear regression. We calculated mean diversity indices of single-state trials and compared the calculated trial diversity indices to those of the respective state.

Results: There were 612 articles reporting pediatric trials during the study period with 565,618 total participants (median per trial 200 [interquartile range 90-571]). Of the 612 articles, 486 (79.4%) reported participant race and 338 (63.4%) reported participant ethnicity. Reporting of participant race increased by 7.9% (95% confidence interval [CI] 0.2-16.3) and of ethnicity increased by 11.4% (95% CI 4.8-18.4) from 2011-2020 (Figure 1). However, the mean annual diversity index of pediatric clinical trials did not change (0.002 percentage points/year, 95% CI -0.007-0.010). Among articles reporting race and ethnicity, the method of assignment was not reported in 51.1% and 71.6% of articles, respectively. Black/African American children were enrolled proportionally more than their U.S. population (odds ratio [OR] 1.88, 95% CI 1.87-1.89). Hispanic/Latino children were enrolled commensurate with their population (OR 1.02, 95% CI 1.01-1.03). American Indian/Alaska Native (OR 0.82, 95% CI 0.79-0.85), Asian (OR 0.56, 95% CI 0.55-0.57), and Native American/Pacific Islander (OR 0.66, 95% CI 0.61-0.72) children were enrolled significantly less compared to their population. White children were enrolled less than expected (OR 0.84, 95% CI 0.84-0.85), though represented 46.0% of participants in trials reporting race/ethnicity. Pediatric clinical trials conducted exclusively in Arizona, Tennessee, Georgia, West Virginia, and New Hampshire had the lowest mean diversity indices and those conducted in California, Maine, Illinois, and North Carolina had the highest diversity indices (Figure 2).

Conclusion: A substantial number of trials in high impact journals did not report participant race/ethnicity, and the method of such assignment was not noted in the majority that did report. Disparities exist in pediatric clinical trial enrollment among American Indian/Native Alaskan, Asian, and Native Hawaiian/Pacific Islander children. The higher representation of Black/African American children compared to U.S. population suggests inclusive research practices that could be extended to other historically disenfranchised racial and ethnic groups.