Our ability to provide safe and effective drug therapy for infants and children is often hampered by a lack of dosing information derived from careful, well-controlled trials and the limited database available concerning drug safety in this patient population. This problem was first articulated 30 years ago by Dr Harry Shirkey from the Children's Hospital in Birmingham, Alabama. He termed infants and children the “therapeutic orphans.”
For the last 3 decades a small group of dedicated and highly motivated pediatricians and pediatric clinical pharmacologists have worked tirelessly to ensure that sick and injured children were able to benefit from the remarkable advances in therapeutics that have characterized the last half of the 20th century. Their efforts have resulted not only in a general level of comfort with pediatric clinical trials but also the passage of legislation designed to ensure that infants and children will benefit from the therapeutic bounty in modern medicine. First, pharmaceutical manufacturers were required to include a pediatric page in their New Drug Application to inform the Food and Drug Administration (FDA) of their intent to develop pediatric indications for a new drug. This was followed by the Pediatric Labeling Rule in 1994, which mandated that all drugs both old and new should have pediatric dosing and safety information in their labels if the drug had potential use for pediatric patients. Finally, the FDA Modernization Act in 1997 provides for a 6-month patent extension for those manufacturers who invest in the development of pediatric safety and dosage guidelines for drugs identified by the FDA as needing such information.
It was in this climate that the members of the Pediatric Pharmacology Research Unit Network along with its administrative oversight group from the National Institute of Child Health and Human Development and selected members of the European Society of Developmental Biology convened a conference in Washington, DC, in 1997 to examine the status of the “therapeutic orphan” problem. Against the background of cautious optimism the participants and their guests discussed in depth the slow progress made during the past 30 years and identified the potential problems facing those who will continue to work for safe and effective drug therapy for infants and children.
The papers presented at the meeting and reproduced for this supplement serve not only as a status report but also to energize those interested to continue their efforts unabated so that the existing legislative promise becomes a reality.