The Need for Pediatric Evaluations
Three quarters of all medications marketed
today do not carry Food and Drug Administration (FDA) approved
labeling for use in neonates, infants, children and adolescents.
Only five of the 80 drugs most frequently used in newborns and
infants are labeled for pediatric use. The FDA has recently made
regulatory changes to facilitate labeling of drugs for pediatric
use. Recent legislative changes in the FDA Modernization Act
(FDAMA) provide strong incentives to conduct studies of drugs
in children. In addition, the FDA 1998 Pediatric rule requires
that drugs with a health benefit for children be studied in children
as well as adults. These two initiatives are encouraging studies
in pediatric patients during the drug development process.
In 1994, the National Institute of Child Health and Human Development (NICHD), initially funded a network of 7 Pediatric Pharmacology Research Units (PPRU) to establish a platform for pediatric clinical trials. The network was expanded to 13 sites in 1999 in response to the increased demand for pediatric clinical studies stimulated by FDAMA and the FDA 1998 Pediatric Rule. The expanded PPRU network provides a unique consortium of pediatric centers to conduct clinical trials and related translational research in pediatric therapeutics.
Past therapeutic mishaps have clearly shown that dosing derived from adult studies cannot be extrapolated to infants and children. For many drugs, developmental, metabolic, and pharmacokinetics/pharmacodynamic and pediatric safety data are needed for their safe and effective use in children.
An overriding consideration for the PPRU pediatric pharmacologists is to delineate the effects of childhood development on the pharmacokinetics of drugs, the influence of age-specific changes in drug disposition and pharmacodynamics, and the interplay between disease states and stage of development.