Essential Drugs for Infants and Children: North American Perspective

Abstract

“Essential Drugs for Children” implies that some drugs are not essential, which of course, is true. It also implies that essential drugs can and should be differentiated from “nonessential” drugs and targeted as a priority for development for pediatric use. With ∼80% of the marketed prescription drugs in the United States not approved by the US Food and Drug Administration (FDA) for use by children, the goal of obtaining approval and labeling for all drugs currently not labeled for use by children is overwhelming. Therefore, drugs currently available must be prioritized for pediatric development so that available resources can be focused on those agents most important for pediatric use. This is a daunting task. How is an essential drug defined? What are the criteria for selecting essential drugs? Is it the volume of use? Is it the number of children impacted by the drug? Is it the indication(s) for use? Is it the severity of the condition for which the drug is indicated? Is it a drug used to treat a life-threatening condition? For the child with bacterial meningitis, the appropriate antibiotic clearly is essential. For the child with HIV infection or cancer, drugs that will impede progression of the disease, prolong life, and mitigate suffering are essential. A drug used to treat a rare genetic disorder is essential for the child affected by that disorder, although it may have minimal impact on the health of the general population of children. In contrast, an effective vaccine will have profound direct benefit for the entire population. A drug used to treat symptomatically the self-limiting pruritus associated with varicella or insect bites may be widely prescribed, although it is used to treat a relatively trivial condition. Each of some 30 odd subspecialties, if queried, undoubtedly would have its list of essential drugs. … Address correspondence to Ralph E. Kauffman, MD, Director, Office of Medical Research, Children's Mercy Hospital, Division of Medical Research, 2401 Gillham Rd, Kansas City, MO 64108.