NIH funding helps with this
The National Institutes of Health (NIH) serves as an important source of funding for clinical trials in children, particularly for diseases such as cancer that are not as prioritized in industry-sponsored clinical trials The Journal of Pediatrics. The study assessed how the number of pediatric clinical trials, as an indicator of industry and NIH research priorities, compares to the burden of childhood disease in the United States. We found that NIH funded important areas of research that are less frequently prioritized by industry, and this is critical to determining new treatments that children need. “ Ansh Goyal, MD, senior...
NIH funding helps with this
The National Institutes of Health (NIH) serves as an important source of funding for clinical trials in children, particularly for diseases such as cancer, which are not as prioritized in industry-sponsored clinical trialsThe Journal of Pediatrics. The study assessed how the number of pediatric clinical trials, as an indicator of industry and NIH research priorities, compares to the burden of childhood disease in the United States.
We found that NIH funded important areas of research that are less frequently prioritized by industry, and this is critical to determining new treatments that children need. “
Ansh Goyal, MD, senior author, surgical resident at the University of Colorado Anschutz Medical Campus
Dr. Goyal was a medical student at Northwestern University Feinberg School of Medicine when he conducted the study.
"Overall, we found that pediatric clinical trials were correlated with how common or severe diseases are in children. However, some diseases were subject to scrutiny by both NIH and industry despite causing high burdens in children," he said. “The burden of disease must be taken into account when setting research funding priorities so that our most vulnerable children are not left behind.”
Researchers evaluated 3,047 clinical trials initiated with industry funding and 1,480 clinical trials initiated with NIH funding from 2015 to 2020.
Endocrine, metabolic, blood, and immune disorders received the highest priority from both industry and the NIH, likely reflecting investments in broader concerns about obesity and other cardiovascular disease.
However, the number of clinical trials for neonatal diseases, congenital birth defects and asthma significantly delayed, which would be expected based on a high disease burden. These were the top three priority areas for both industry and NIH.
“Pediatric research on asthma and lung disease has been chronically underfunded,” said Susanna McColley Susanna McColley, MD, pulmonary researcher and researcher at Ann & Robert H. Lurie Children’s Hospital in Chicago and professor of pediatrics at Northwestern University Feinberg School of Medicine. “We need increased funding for institutions like the National Heart, Lung and Blood Institute (NHLBI), with dedicated funding and dedicated research allocations to address research gaps and promote rapid innovation in these areas.”
The authors find that the NIH provides essential information and transparency on federal investment priorities because they are funded by U.S. taxpayers. In contrast, challenges related to data availability, transparency, and ownership of industry-sponsored studies may limit the depth and scope of industry-specific analyzes regarding research prioritization.
“Clinical trials are essential to advancing pediatric healthcare,” said Matthew Davis, MD, Mapp, physician-in-chief and chief scientific officer at Nemours Children’s Health, lead scientist on the study. "Ultimately, the combination of NIH and pharmaceutical industry support for pediatric clinical trials will strongly shape the way clinicians can help children combat health challenges and grow into healthy adults as possible. Focusing scientific efforts on conditions that consistently account for a high burden of disease will have the greatest impact."
Sources:
Goyal, A., et al. (2025) Pediatric Burden of Disease in the United States, 2015-2020. The Journal of Pediatrics. doi.org/10.1016/j.jpeds.2025.114525.