NIH funding helps address pediatric disease gaps in clinical trials

The National Institutes of Health (NIH) serves as an important source of funding for clinical trials in children, especially for diseases, such as cancer, that are not as highly prioritized in industry-sponsored clinical trials, according to a recent study published in The Journal of Pediatrics. The study evaluated how the number of pediatric clinical trials, as an indicator for industry and NIH research priorities, compared with the burden of childhood disease in the United States.

We observed that NIH funded key research areas that are less often prioritized by industry, and this is critical for establishing new treatments that children need."

Ansh Goyal, MD, lead author, surgical resident at the University of Colorado Anschutz Medical Campus

Dr. Goyal was a medical student at Northwestern University Feinberg School of Medicine while conducting the study.

"Overall, we found that pediatric clinical trials were correlated with how common or severe diseases are for children. But some diseases were under-prioritized by both NIH and industry, despite causing high burden to children," he said. "Disease burden needs to be considered when setting research funding priorities so that our most vulnerable children are not left behind."

Researchers assessed 3,047 clinical trials that were initiated with industry funding and 1,480 clinical trials initiated with NIH funding from 2015 through 2020. They found that the leading 20 disease categories with the greatest burden of disease were the focus of less than half of all clinical trials, whether funded by industry (43 percent) or by NIH (42 percent).

Endocrine, metabolic, blood and immune disorders received highest prioritization by both industry and the NIH, likely reflecting investments in broader obesity concerns and other cardiovascular conditions.

The number of clinical trials for neonatal disorders, congenital birth defects and asthma, however, significantly lagged what would be expected based on high disease burden. These were the top three least prioritized areas by both industry and the NIH.

"Pediatric research in asthma and lung diseases has been chronically under-funded," said co-senior author Susanna McColley, MD, pulmonologist and researcher at Ann & Robert H. Lurie Children's Hospital of Chicago and Professor of Pediatrics at Northwestern University Feinberg School of Medicine. "We need increased funding to institutions such as the National Heart, Lung, and Blood Institute (NHLBI) with special calls for funding and special research allocations to help address research gaps and promote rapid innovation in these areas."

The authors note that the NIH provides substantive information and transparency regarding federal investment priorities, as they are funded by U.S. taxpayers. In contrast, challenges related to data availability, transparency, and the proprietary nature of industry-sponsored trials can limit the depth and scope of industry-specific analyses regarding prioritization of research.

"Clinical trials are essential to advancing healthcare for children," said Matthew Davis, MD, MAPP, Enterprise Physician-in-Chief and Chief Scientific Officer at Nemours Children's Health, senior scientist for the study. "Ultimately, the combination of support from the NIH and the pharmaceutical industry for pediatric clinical trials will strongly shape how clinicians are able to help children battle health challenges and grow into as healthy adults as possible. Focusing scientific efforts on conditions consistently responsible for high levels of disease burden will achieve the greatest impact."