O3245: Racial and Ethnic Diversity in Pediatric Clinical Trials for Diabetes and Obesity: A Review of Trials Submitted to the US Food and Drug Administration (FDA) under the Pediatric Research Equity Act (PREA) from 2005-2023

Background: The US FDA encourages diversity in clinical trials, including diversity of race and ethnicity. Racial/ethnic minorities are often underrepresented in adult and pediatric clinical research, which creates knowledge gaps about safety and efficacy in these groups and may negatively impact patient acceptance. Furthermore, diseases such as asthma, diabetes, HIV/AIDS, hypertension, obesity, and sickle cell disease are known to disproportionately affect racial/ethnic minorities. This study examined whether children from racial/ethnic minorities are underrepresented or overrepresented in pediatric clinical trials compared to the national representation of racial/ethnic subgroups for select diseases.

Methods: We conducted a cross-sectional study on racial/ethnic diversity of enrollees in diabetes and obesity pediatric clinical trials (US only and global) submitted to the US FDA under the Pediatric Research Equity Act (PREA) from 2005 to 2023. Race/ethnicity data were obtained from FDA clinical reviews and sponsor-submitted clinical study reports. Expected pediatric US population with diabetes and obesity were estimated using census data from the American Community Survey (ACS) and prevalence estimates for diabetes and obesity from the SEARCH for Diabetes in Youth study and the National Health and Nutrition Examination Survey (NHANES) respectively. We included only trials with complete data on race (diabetes, n=14; obesity, n=3) and ethnicity (diabetes, n=12; obesity, n=3) in the final analysis. Chi-square goodness of fit tests (α=0.05) were used to compare the observed proportions of trial enrollees with the expected proportions of US children with diabetes and obesity by race and ethnicity.

Results: Compared to the racial/ethnic distribution of the US pediatric population with diabetes, there was strong evidence of underrepresentation (p < 0.05) by race in 9 out of 14 and by ethnicity in 9 out of 12 of the diabetes trials included in our study (Table 1). These trials were mostly efficacy trials with labelling approval dates after 2015 (n=6). American Indian/Alaskan native children were the most underrepresented race, followed by Black or African American, and Asian and Native Hawaiian/Pacific Islander children. Children of Hispanic ethnicity were mostly underrepresented in the trials studied. For obesity, there was strong evidence of underrepresentation (p < 0.05) by race and ethnicity in 1 out of 3 trials compared to the race/ethnicity distributions of the US pediatric population with obesity (Table 2). This trial was an efficacy trial with labelling approval date after 2015. Children of Asian race and Hispanic ethnicity were underrepresented in all obesity trials studied.

Conclusion: Our study provides important insights into the representation of US racial and ethnic minorities in pediatric clinical trials for diabetes and obesity. To our knowledge, this is the first study looking at racial/ethnic enrollment for these diseases in children. Our findings highlight the need for continued efforts to ensure equitable participation of minorities in clinical research especially in safety and efficacy studies.