P4A085: Pediatric Industry and Nih-funded Clinical Trials vs Pediatric Burden of Disease in the United States from 2015-2020

Background: Previous studies using publicly available data have demonstrated NIH-funding for US clinical trials, as well as global clinical research activity, is moderately associated with pediatric burden of disease. However, characterizing industry investments across all disease categories has been difficult due to lack of available data. As more pediatric clinical trials are sponsored by industry instead of the government, there is a need to elucidate industry priorities and their association with national burden of disease. The objective of this study is to evaluate the number of pediatric clinical trials from 2015-2020 as a proxy for industry and NIH research priorities and compare them to national burden of childhood disease during the same period.

Methods: We conducted a cross-sectional five-year analysis of industry and NIH-funded pediatric clinical trials between January 1, 2015, and January 1st, 2020. Data on disease burden (by DALYs, YLL, YLD and deaths) was extracted from the Institute of Health Metrics and Evaluation (IHME). Advanced searches with IHME categories of disease for pediatric ( < 18yrs) interventional clinical trials in the US initiated in 2015-2020 were completed on Clinicaltrials.gov. Descriptive statistical analysis was used to determine the association between clinicals trials and US burden of disease.

Results: Industry funded pediatric clinical trials had a weak linear relationship with DALYs (R squared = 0.006, Figure 1A) and a moderate monotonic relationship (Spearman rank = 0.58, Table 1). NIH-funded clinical trials had a weak linear relationship with DALYs (R squared = 0.005, Figure 1A) and a weaker monotonic relationship (Spearman rank =0.44, Table 1). The top 3 disease categories by DALYs with no pediatric clinical trials funded by industry in the last 5 years were SIDS, gynecological diseases, and neck pain. The top 3 disease categories by DALYs with no clinical trials funded by the NIH in the last 5 years were low back pain, viral skin diseases, and lower respiratory infections. Based on expected number of clinical trials by DALYs assuming a linear relationship with burden of disease, the least prioritized disease categories by both industry and NIH were neonatal disorders, congenital birth defects, and asthma (Figure 1B, C). The top 3 most prioritized categories were blood, metabolic and immune disorders, other neurological disorders, and other sense organ (eye, ear, tongue) diseases for industry-funded, and blood, metabolic and immune disorders, other neurological disorders, and HIV/AIDS for NIH-funded studies (Figure D, E).

Conclusion: Industry-funded pediatric clinical trials in the US are moderately correlated with pediatric disease burden across multiple measures of disease including DALYs, deaths, YLLs, and YLDs. Given differing motivations for investment between government and industry sources of funding, under-prioritized pediatric diseases with significant burden of disease deserve special consideration for clinical trials or deserve to be highlighted and incentivized through future legislative action.