OPPTB Research Programs

Multiple knowledge gaps regarding the use of therapeutics in children and pregnant and lactating people have resulted in inadequate labeling and frequent off-label use of prescription drugs in these populations. NICHD—particularly OPPTB—supports pharmacokinetic (PK), pharmacodynamic (PD), and pharmacogenomic (PG) research on medications for children and pregnant and lactating people to help ensure their safety and usefulness for preventing, treating, and managing diseases and disorders.

This focus and mission include Best Pharmaceuticals for Children Act (BPCA) activities, as well as the T32 Pediatric Clinical and Developmental Pharmacology Training Network.

Specific areas of interest within pediatric and pregnant and lactating populations include:

• Developmental pharmacology and toxicology
• Development of formulations and devices appropriate for pediatric use
• Development of formulations and devices appropriate for use in pregnant and lactating people
• Safety and PK studies
• PD studies
• PG and personalized medicine
• Identification of molecular targets and development of targeted medications in diseases that affect populations of interest
• Identification of biomarkers appropriate for pediatric use
• Innovative study designs for small populations
• Preclinical and nonclinical models of drug disposition and effect
• Mechanisms of drug effects (efficacy and safety)
• Mechanisms of changes in PK and PD over time, including throughout gestation and childhood development

Program Official: Antonello Pileggi

In the care of infants and young children who need implanted medical devices, the fact that the children grow but the devices do not is an ongoing challenge. Thus, children receiving devices may need multiple surgeries for revision as they grow. 3D printing, a form of additive manufacturing, potentially may provide a solution to address this need. 3D printing builds up parts layer-by-layer from a digital model from metals, plastics, or even ceramics and has the potential to produce complex biomedical devices according to computer design using patient-specific anatomical data. This process allows complex devices to be matched to a patient's anatomy. Most importantly, because 3D printing does not require specialized cutting tools or molds, design changes can be made very rapidly and applied to only a small number of units. RFA-HD-15-023 and RFA-HD-15-024 invited applications that will lead to development of minimally invasive/non-implantable flexible medical devices. The purpose of RFA-HD-19-001: Safe and Effective Devices for Use in Neonatal, Perinatal, and Pediatric Care Settings (R43/R44 Clinical Trial Optional) is to lead the development of implantable devices that will adapt to the needs of growing children.

Topic areas for this research include:

• Identification of a specific clinical problem in children that will be solved by device implantation where the device needs to adapt to the child's growth
• Development of 3D-printed biodegradable scaffolds appropriate to the clinical problem
• Testing of the scaffold in long-term animal models