The pediatric studies initiative: after 15 years have we reached the limits of the law?

Clin Ther. 2014 Feb 1;36(2):156-62. doi: 10.1016/j.clinthera.2013.11.007.


Background: Despite considerable disincentives for conducting drug studies in children, 15 years ago the Food and Drug Administration, pediatric health advocates and congressional sponsors created a carrot-and-stick policy approach of voluntary and mandatory programs to encourage the pharmaceutical industry to include children in the drug development process. After several rounds of reauthorization of the laws on a temporary basis, the enabling statutes have been made permanent.

Objective: The purpose of this analysis is to review the advances that resulted from the law and the areas where further progress is needed.

Methods: A brief review of the history and results of the pediatric studies initiative was conducted by the authors and a determination made about the accomplishments of the law and remaining challenges.

Results: Indicators of the changes that resulted from this pediatric studies initiative are both indirect, such as the increase in the number of indication supplements for new populations, and direct, such as the decrease in the percentage of medicines used off-label in children. Although the pediatric studies initiative has significantly improved therapeutic options for children, concern still exists that drug companies are reluctant to include children in drug development unless continuously incentivized, whether positively or negatively. Two challenges are particularly problematic: neonatal studies and child-friendly formulations.

Conclusion: Although the latest round of legislation should provide opportunities to address these problems, significantly more effort will be needed to achieve real culture change. Ultimately, the solution will require full program implementation by the Food and Drug Administration and close collaboration by many key stakeholders to ensure that pediatric studies become a routine part of the drug development process.

Keywords: carrot and stick; incentives; neonates; pediatric formulations; pediatrics; research and development.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Chemistry, Pharmaceutical / legislation & jurisprudence*
  • Drug Discovery / trends*
  • Drug Industry* / trends
  • Humans
  • Infant, Newborn
  • Pediatrics / legislation & jurisprudence*
  • United States
  • United States Food and Drug Administration / legislation & jurisprudence