Status of drug approval processes and regulation of medications for children.

The term therapeutic orphan was coined in 1968 to describe the exclusion of infants and children from approved indications for use of the majority of drugs approved by the US Food and Drug Administration (FDA). Although the 1962 Kefauver-Harris amendments to the Food, Drug, and Cosmetic Act were designed to insure the efficacy and safety of drugs approved for human use, infants and children have largely been excluded from the protection of the law. Approximately 80% of the drugs approved by the FDA during the past 30 years have been approved with a labeling disclaimer for use by children. This high percentage is due largely to the lack of studies in children to document safety and efficacy or a failure to use available data to amend labeling to include pediatric indications. Recently, several initiatives by the FDA and the National Institute of Child Health and Human Development have been implemented that promise to increase the number of drugs studied and labeled for children. These initiatives may introduce a new era of drug development for children in which pediatric investigators, the FDA, the National Institutes of Health, and the pharmaceutical industry join together to bring the same level of pharmacotherapeutic safety and efficacy to children that adult patients enjoy.