Part of the BPCA mandate is to collaborate with experts in pediatric research and care to determine knowledge gaps in pediatric therapeutics. In addition to directly sponsoring clinical trials, BPCA also cosponsors important pediatric therapeutic activities and initiatives with other NIH institutes organizations.
Through the Research in Pediatric Developmental Pharmacology Centers Program (RPDP), NICHD funds research centers to provide an arena for multidisciplinary interactions between basic and clinical scientists interested in establishing translational research programs in pediatric developmental pharmacology. The centers also serve as a national resource for the education of new scientists (U54 trainees) electing to pursue careers in translational research in high priority areas of pediatric developmental pharmacology. Center investigators also facilitate important community outreach and education efforts to increase awareness and convey the importance and implications of their research activities to the general public.
The mission of the Biomarkers in Pediatric Therapeutics Special Interest Group is to pursue opportunities for strengthening cross-disciplinary pediatric biomarker research at NIH while innovating beyond existing investments. Its goals are to provide leadership, vision, and support to promote a strong body of pediatric biomarker research funded by NIH; and to collect, evaluate, and disseminate scientific information and funding opportunities for biomarker research in pediatric therapeutics at NIH. The group promotes trans-NIH funding announcements, hosts speakers, and support panels and minisymposia, national and international webinars, and group discussions.
For more information, visit https://oir.nih.gov/sigs/biomarkers-pediatric-therapeutics-scientific-interest-group.
The lack of a pediatric formulation was one consideration for prioritization because the lack of commercially available oral pediatric formulations is a continuing problem for children, parents, and pediatricians. Also, there is no coherent approach to the development of pediatric formulations.
The ideal pediatric dosage form would:
- Be orally dissolvable
- Be tasteless
- Have an appropriate dosage increment for the smallest infants
- Contain minimal amounts of inactive substances like fillers or preservatives
- Be stable in light, humidity, and heat
- Have the necessary release characteristics, as applicable
The Pediatric Formulations Platform uses NIH BPCA funds to fund FDA chemists' assessment of pediatric product formulations and produce an open-source, publicly available approach to pediatric oral formulations manufacturing.
The work includes five tasks:
- Perform an assessment of all commercially available products to determine which have pediatric formulations.
- Determine what technologies are publicly available, how these technologies have been used, and for what types of products.
- Use prototypical drug products and computational methods to distinguish their molecular structure through characteristics such as solubility, permeability, light sensitivity, pH instability, heat instability, hygroscopic properties, and bitterness.
- Determine the best formulations technology for specific drug categories based on information from tasks 1–3.
- Produce prototype batches of selected drug products.
The goal is to make these data publicly available as soon as possible.
As a first step, the Biopharmaceutics Classification System assessment of all commercially available products is archived as "Oral Formulations Platform—Reports 1 and 2." When further stages are completed, they will be posted.
- Oral Formulations Platform—Report 1 (PDF 748 KB)
- Oral Formulations Platform—Report 2 (PDF 1 MB)
- NICHD-Funded Research Finds Biological Basis for Children's Taste (PDF 334 KB)
- A Report from the Pediatric Formulations Task Force: Perspectives on the State of Child-Friendly Oral Dosage Forms (PDF 475 KB)
BPCA formed a working group to discuss the current state of hypertension epidemiology, pathophysiology, diagnosis, and treatment in the pediatric population. Clinicians have grappled with the issue of treatment versus non-treatment for pediatric hypertension, and recent studies have shown that only a small percentage of children with hypertension are treated for the condition.
The working group published a white paper summarizing issues and gaps identified in this area and determining key areas for future research.
- Taylor-Zapata, P., Baker-Smith, C.M., Burckart, G., Daniels, S.R., Flynn, J.T., Giacoia, G.,…Zajicek, A. (2019). Research Gaps in Primary Pediatric Hypertension. Pediatrics, 143 (5) e20183517; DOI: https://doi.org/10.1542/peds.2018-3517
A working group meeting was held on September 25, 2017.