The need for pediatric drug development goes back more than a century. In the early 1900s, elixirs used to help children sleep led to unintended tragedies and death. Later, in the 1950s and 1960s, thalidomide prescribed to pregnant people for nausea resulted in birth defects, including limb deformities.
The U.S. Food and Drug Administration’s Pediatric Rule of 1994 allowed the labeling of drugs for pediatric use based on extrapolation of efficacy in adults and additional pharmacokinetics (how a drug affects the body), pharmacodynamics (how the body affects a drug), and safety studies in pediatric patients, if the course of the disease and the response to the drug are similar in children as in adults.
BPCA legislation of 2002 directed the Secretary of Health and Human Services, acting through the NIH Director, to establish a program for pediatric drug development. The NIH Director delegated lead responsibility for BPCA’s off-patent drug component to NICHD.
NICHD was chosen to lead NIH's BPCA efforts, in part, because of its success with the now-retired Pediatric Pharmacology Research Unit (PPRU) Network. The PPRU comprised a group of experts in pediatric pharmacology who conducted pediatric clinical trials from 1994 to 2009. Their work initiated key pediatric research, including clinical trial designs, and many label changes for drugs used in children. In addition, NICHD's strengths in sponsoring research and conducting safe and effective clinical trials in populations thought to be “fragile,” such as children and pregnant people, made the institute the natural choice to spearhead NIH's BPCA activities.
From 2003 through 2009, NIH funded academic centers to conduct large dosing, safety, and efficacy studies. In 2010, NICHD funded the Pediatric Trials Network as the infrastructure for the conduct of clinical trials that lead to label changes for off-patent medications used in children.