Off-label Drug Use in Hospitalized Children | Clinical Pharmacy and Pharmacology | JAMA Pediatrics | JAMA Network
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March 2007

Off-label Drug Use in Hospitalized Children

Author Affiliations

Author Affiliations: Division of Infectious Diseases, Children's Hospital of Philadelphia, Philadelphia, Pa, and the Center for Clinical Epidemiology and Biostatistics, University of Pennsylvania School of Medicine, Philadelphia (Dr Shah); Child Health Corporation of America, Shawnee Mission, Kan (Dr Hall and Mr Bertoch); Department of Pediatrics, Feinberg School of Medicine, Northwestern University, and Division of Pediatric Critical Care Medicine, Children's Memorial Hospital, Chicago, Ill (Dr Goodman); Division of Pediatric Critical Care Medicine (Dr Feuer) and Ambulatory Care Network, and Phyllis and David Komansky Center for Children's Health (Dr Hyman), Morgan Stanley Children's Hospital of New York–Presbyterian Hospital, Columbia University Medical Center, New York, NY; Department of Pediatrics, Children's Mercy Hospitals and Clinics, Kansas City, Mo (Dr Sharma); Division of General Pediatrics (Dr Fargason) and Divisions of Critical Care and Emergency Medicine, Department of Pediatrics (Dr White), University of Alabama at Birmingham; Department of Cardiology, Children's Hospital, Boston, Mass (Dr Jenkins); University of Texas Southwestern Medical School and Children's Medical Center, Dallas (Dr Levy); Children's Hospitals and Clinics of Minnesota, St Paul (Dr Levin); and Children's Research Institute, Children's National Medical Center, Washington, DC (Dr Slonim). Dr Levin is now with Children’s Hospital of Pittsburgh, Pittsburgh, Pa.

Arch Pediatr Adolesc Med. 2007;161(3):282-290. doi:10.1001/archpedi.161.3.282

Objectives  To describe the magnitude of off-label drug use, to identify drugs most commonly used off-label, and to identify factors associated with off-label drug use in children hospitalized in the United States.

Design  Retrospective cohort study.

Setting  Administrative database containing inpatient resource utilization data from January 1 to December 31, 2004, from 31 tertiary care pediatric hospitals in the United States.

Participants  Hospitalized patients 18 years or younger.

Main Exposures  Institution and patient characteristics.

Main Outcome Measures  Off-label drug use was defined as use of a specific drug in a patient younger than the Food and Drug Administration–approved age range for any indication of that drug.

Results  At least 1 drug was used off-label in 297 592 (78.7%) of 355 409 patients discharged during the study. Off-label use accounted for $270 275 849 (40.5%) of the total dollars spent on these medications. Medications classified as central or autonomic nervous system agents or as fluids or nutrients, or gastrointestinal tract agents were most commonly used off-label, whereas antineoplastic agents were rarely used off-label. Factors associated with off-label use in multivariate analysis were as follows: undergoing a surgical procedure, age older than 28 days, greater severity of illness, and all-cause in-hospital mortality.

Conclusions  Most patients hospitalized at tertiary care pediatric institutions receive at least 1 medication outside the terms of the Food and Drug Administration product license. Substantial variation in the frequency of off-label use was observed across diagnostic categories and drug classes. Despite the frequent off-label use of drugs, using an administrative database, we cannot determine which of these treatments are unsafe or ineffective and which treatments result in substantial benefit to the patient.