Therapeutic orphans.

By an odd and unfortunate twist of fate, infants and children are becoming “therapeutic or pharmaceutical orphans.”1 Since 1962 they have been denied the use of many new drugs. The Drug Laws of 1962 had their inception following a pediatric tragedy—the thalidomide catastrophe. The laws of 1938 followed another which resulted from the use of a pediatric dosage form, “elixir” of sulfanilamide. By “legal” definition, drugs introduced since 1962 must be safe and efficacious, but only a small number of these have been studied in the pediatric age group. Certainly, there are some drugs which have an anticipated use only in adults; it would be unreasonable to ask for certification of these for use in infants and children. There are a small number of new drugs, released since 1962, which had an anticipated use for infants and children as well as for adults, and their applications for approval have been passed after the required studies in pediatric and adult patients. However, many of the drugs released since 1962 carry an “orphaning” clause, eg, “Not to be used in children … is not recommended for use in infants and young children since few studies … Address correspondence to Harry Shirkey, MD, Department of Pediatrics, Children's Hospital, Birmingham, AL.