‘Off Label’ Drug Use in Children

Saleh F. Ajlouni


Three quarters of all medications marketed today do not carry Food and Drug Administration (FDA) approved labeling for use in neonates, infants, children and adolescents. This has been stated by the pediatric pharmacology research unit Network which is established by the national institute of Child Health and Human Development in the United States. It is shocking to note that few drugs, only approximately 20% of all drugs marketed in the United States, have been labeled for use by infants and children. Eighty percent or more of drugs approved since 1962 have been approved and labeled for use in adults with a disclaimer in the labeling that they are not approved for use by children (as indicated by the American Academy of Pediatrics in September 12,1996). Many commercially available drugs are only licensed for use in adults and are not used according to the product licensing in pediatric practice.
Only minorities of the currently marketed drugs have undergone pediatric clinical trials and have approved labeling for use in children. These include common antimicrobial agents, medications for fever, vaccines and some asthma and allergy medications. However, most drugs used to treat illnesses in children have never been formally tested or approved for pediatric use and lack even basic dosage recommendations for children in their labeling. These include such routinely used medications as dopamine (used to treat shock), cisapride (used to treat abnormal regurgitation of stomach contents in infants and small children), ketorolac (the only available injectable non-narcotic pain reliever), midazolam (used as a sedative and to treat convulsions) and adenosine (used to treat life threatening abnormal heart beats) and the list is much longer.
The treatment of pediatric patients with drugs in hospitals is being impeded by a shortage in the availability of licensed drugs in an appropriate formulation. There are several reasons for this situation, ethical problem of research in children, the reluctance of parents to allow their children to participate in drug trials and the technical challenges small study participants bring along. Possibly because of the underestimation of the problem, there is a lack of funding from governments, health care providers and industry. As a result, pediatric drug trials are relatively scarce and in many cases contain only a limited number of patients. The purpose of licensing is to ensure that medicines are examined for safety, efficacy and quality. Most medicines administered to adults have a product license that outlines the particular indication, dose and route of administration for a drug. However, many medicines used for children are not licensed for use in children or are used outside the terms of product license (‘off label’). This means that the risks or benefits of using a drug in that particular situation have not been examined by the licensing authority.
The aim of this review is to emphasize the need to make studies and to focus on dosing and safety data for children in an appropriate pediatric population a requirement during clinical trials of each new drug with potential use by children.
This review also discusses the operation of the licensing system and the use of unlicensed and off-label drugs in children.


Medicine Act (MCA), Proved License (PL), European (EU), Marketing Authorization (MA), Food and Drug Administration (FDA).

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