Pediatric Burden of Disease in the United States, 2015-2020.

Objective: To evaluate how the number of pediatric clinical trials, as a proxy for industry and National Institutes of Health (NIH) research priorities, compared with population-level burden of childhood disease in the United States (US).

Study Design: Cross-sectional study evaluating associations between initiated clinical trials funded by industry or the NIH and pediatric burden of disease in the US. Disease burden was measured as deaths, disability-adjusted life-years (DALYs), years of life lost (YLL), and years lived with disability (YLD).

Results: From 2015 through 2020, 3047 clinical trials were initiated with industry funding, compared with 1480 clinical trials initiated with NIH funding. The leading 20 disease categories with the greatest burden of disease by DALYs accounted for 79.4% of total DALYs; these 20 categories were the focus of 43.5% of all industry-funded trials and 41.9% of NIH-funded trials during the study period. Industry-sponsored pediatric clinical trials had a moderately strong monotonic relationship with DALYs (r=0.59), compared with r=0.44 for NIH-funded clinical trials. Disease burden as measured by deaths, YLLs, and YLDs demonstrated a broad range of correlations with clinical trial initiation, with r values ranging from 0.18 to 0.48. The least prioritized high-burden disease categories by both industry and NIH were neonatal disorders, congenital birth defects, and asthma.

Conclusions: Industry-sponsored and government-funded pediatric clinical trials in the US are variably associated with pediatric disease burden across measures of disability and mortality. Under-prioritized pediatric diseases with significant burden of disease deserve special consideration for clinical trials through future legislative attention and corresponding funding.