Table 1

Key milestones in pediatric product development.

1977American Academy of Pediatrics statement on the need for clinical trials in children.
1979FDA rule: Pediatric Use Section established in package labels.
1983Orphan Drug Act signed, which provided federal assistance in the development of products for the diagnosis, treatment, and prevention of rare diseases/conditions. Health and Human Services (HHS) Final Rule incorporated protection of children as research subjects in HHS research.
1994Modification of the FDA rule related to Pediatric Use and extrapolation of adult data; Required that a statement be included in the drug labels of agents for which there is not sufficient evidence that products are appropriate for pediatric use.
1997Food and Drug Administration Modernization Act (FDAMA): 6 months of extended patent protection is given for sponsors who provide certain pediatric data in compliance with the FDA.
1998Public Health Service/NIH policy requiring inclusion of children in clinical research. Pediatric Rule: required sponsors to assess pediatric use for new drug applications.
2000Children’s Health Act required research on FDA-regulated pediatric products to comply with HHS subpart D and a review of subpart D by the secretary of HHS.
2001International Conference on Harmonisation (Clinical Investigation of Medicinal Products in the Pediatric Population) came into operation.
2002Pediatric Rule was enjoined as a result of a legal challenge. BPCA: renewed pediatric exclusivity provisions and provided for testing of on- and off-patent drugs in children; a Priority List was developed by FDA in collaboration with NICHD.
2003PREA: required the study of drugs and biologics in children with only specific exceptions; established the Pediatric Advisory Committee.
2006NIH Modernization Act: established the CTSA consortium in the United States and preserved infrastructure and funding for pediatric centers.
2007The Food and Drug Administration Amendments Act (FDAAA): reauthorized BPCA and PREA and included the Pediatric Medical Device Safety and Improvement Act European Union Pediatric Regulation: Comprehensive regulation that required that pediatric data be available at the time of drug approval based on a pediatric investigation plan.

Adapted from: S. Maldonado, C. Rose, S. Conttrell, B. Goldstein, Regulatory guidelines for pediatric drug development: Stimulation for regulatory drug research by regulatory authorities, in Pediatric Drug Development, Concepts and Applications, A. E. Mulberg, S. A. Silber, J. van den Anker, Eds. (Wiley, New Jersey, 2009), pp. 133-171.