There is a major need to integrate clinical pharmacology and disease-specific therapeutic issues—identification of similarities and differences in the etiology, diagnosis, pathophysiology, prognosis, and natural response to therapy—of diseases or conditions affecting children and adults. The NICHD and BPCA partly address this need through the IAPT.
The initiative focuses on identifying similarities and differences in diseases or conditions expressed in children and adults by studying:
- Diagnostic biomarkers
- Outcome Measures/Endpoints
- Manifestation (Phenotypic Expression)
- Natural History
- Response to Therapy
- Extrapolation Issues
Asthma, psychiatry, and diabetes were chosen as prototype therapeutic areas for the IAPT, and working groups were established for each area in 2012. Chairs of the working groups convened at the 2012 Workshop on Advancing Pediatric Therapeutics (PDF 126 KB) to formally launch the initiative. An additional group was added later to work toward advancing the ontogeny of transporters in children. Outcomes for the working groups include identifying knowledge gaps and research needs between clinical pharmacology and disease-specific therapeutic issues.
The asthma working group's goal was to develop a white paper for publication. It accomplished this goal in the Journal of Allergy and Clinical Immunology's (JACI) January 2014 theme issue , which covered "asthma across the ages."
The diabetes working group had a similar goal and in 2016, published "Expanding Treatment Options for Youth with Type 2 Diabetes: Current Problems and Proposed Solutions: A White Paper from the NICHD Diabetes Working Group" in Diabetes Care.
Working Group to Advance Ontogeny of Drug Transporters in Children
This working group includes researchers dedicated to the study of drug transporters with the goal of promoting this area of research to determine their role in drug distribution and effects, including toxic effects. The specific objectives are to promote knowledge creation, develop a roadmap of research needs and priorities, and motivate other researchers from necessary disciplines.
Initial activities included compiling feedback received from experts about what research is currently being conducted and problems encountered in studying ontogeny of transporters/receptors.
The group's Literature Review subgroup developed a literature search strategy for the ontogeny of transporters in certain organs, using transporters identified by the International Transporter Consortium. This subgroup, which was further divided into three groups that addressed the ontogeny of transporters in the liver, intestine, and kidneys, published a white paper in 2015 in Clinical Pharmacology and Therapeutics.
The PFI is designed to look at all the factors that affect the development of pediatric drugs. The goals are to create a roadmap for removing/mitigating barriers to pediatric drug development and to connect researchers and experts in pediatric formulation to discuss issues and challenges that may stimulate further research in the field.
The PFI has four groups:
- Scientific, technical, and regulatory barriers for the development of pediatric formulations
- Taste, smell, and flavor research in infants and children
- Economic issues and partnerships
- Use and application of new drug delivery systems in pediatrics
The following are links to the minutes from past PFI meetings:
- November 1–2, 2011
Bolger Center, Potomac, MD
Meeting Minutes for Pediatric Formulations Initiative (PFI) Workshop November 2011 (PDF 474 KB)
- December 6–7, 2005
Bethesda Marriott, Bethesda, MD
Meeting Minutes for Pediatric Formulations Initiative (PFI) Working Meeting December 2005 (PDF 250 KB)
This collaboration was an interagency agreement between NICHD and NHLBI's PROP Network to investigate the molecular mechanisms that contribute to respiratory disease risk of premature neonates over the first year after birth.
Pryhuber, G.S., Maitre, N.L., Ballard, R.A., Cifelli, D., Davis, S.D., Ellenberg, J.H.,…Hamvas, A., and The Prematurity and Respiratory Outcomes Program Investigators. (2015). Prematurity and respiratory outcomes program (PROP): study protocol of a prospective multicenter study of respiratory outcomes of preterm infants in the United States. BMC Pediatr, 15, 37. PMID: 25886363