Food and Drug Administration approval for medications used in the pediatric intensive care unit: a continuing conundrum.

Objectives: Many drugs used in the pediatric intensive care unit are administered "off label," i.e., they have been neither thoroughly tested for efficacy and safety nor approved for use in children. The U. S. Congress has enacted legislation to promote standards and requirements for Food and Drug Administration labeling for drugs used in pediatrics. Nevertheless, we hypothesized that most medications used in our pediatric intensive care unit were not Food and Drug Administration approved for use in pediatric patients. Design: A list of medications dispensed in the pediatric intensive care unit from January through February 2008 was obtained from our pharmacy database. We then determined whether each medication had been granted Food and Drug Administration approval for use in children. Medications were divided into the following categories: not approved for use in any pediatric age group, approved for use in limited age groups only, and approved for use in all pediatric age groups. Setting: A pediatric intensive care unit at a tertiary care hospital with 26 beds and 1,500 admissions per year. Measurements and Main Results: In the 2-month period, 248 different medications were dispensed with a total of 49,707 medication orders. Sixty (24.2%) of the medications dispensed were not Food and Drug Administration approved for use in any pediatric age group, 106 (42.7%) were approved for use in limited age groups, and 82 (33%) were approved for use in all pediatric age groups. Eleven of the 25 most frequently dispensed medications were approved for use in limited age groups, but none of them was used for the indication or age group for which they were approved. Conclusions: Despite the efforts of Congress, 67% of medications prescribed and administered in the pediatric intensive care unit did not have Food and Drug Administration approval or had only limited approval, underscoring the need for the medical community to demand oversight and research to improve drug labeling for our patient population. (Pediatr Crit Care Med 2011; 12: e195-e199)