Pediatric "Off-Label" Prescribing: What Every APN Should Know

Surprisingly few medications routinely prescribed for children have actually been studied for pediatric use. Medications that are safe for adults may have detrimental effects on children. However, the trend of forgoing pediatric drug research continues. The lack of pediatric data leads to "off-label" prescribing by health care providers, although most advanced practice nurses follow specific prescription protocols. The history and scope of "off- label" prescribing and its effects on the general public are discussed. The need for pediatric clinical trials is addressed as well as the FDA's attempts to improve the safety of pediatric drug therapy. 2) though most APNs follow set protocols. This article examines the issues related to children being treated with medications that have not been approved by the FDA for pediatric use. The history of pediatric dosing, the obstacles to change and the risk to benefit assessment of unapproved use of medications are investigated. The current perspectives, professional responsibilities, effects on the health care industry, and recommendations for change are also discussed. HISTORY In the United States, a dilemma currently exists whereby children are commonly treated with drugs that have not been approved for pediatric use by the FDA. In 1962, the Kefauver-Harris amendments to the Food, Drug, and Cosmetic Act were designed to ensure safety and effectiveness for human drug use. However, infants and children were excluded from the protections that these amendments were to provide. Once a drug is approved for use by the FDA further studies to determine safety and efficacy in infants and children are rarely conducted.( 3) The issue of the lack of pediatric drug data is important because effective treatment may be withheld or children may be treated with medications without a full understanding of the risks, benefits, and implications. How many parents realize that their child is treated with medications whose safety has not been established?