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Off-label drug use in hospitalized children
Samir S Shah1, Matthew Hall, Denise M Goodman
1Division of Infectious Diseases, Children's Hospital of Philadelphia, 34th Street & Civic Center Boulevard, Philadelphia, PA 19104, USA. shahs@email.chop.edu
Insights
Most hospitalized children in the US receive off-label drug prescriptions, particularly central nervous system agents. This study identified factors associated with off-label medication use in pediatric inpatients.
Area of Science:
- Pediatric pharmacology
- Drug utilization research
- Health services research
Background:
- Off-label drug use is common in pediatric populations due to limited FDA-approved medications for children.
- Understanding the scope and patterns of off-label prescribing is crucial for patient safety and effective treatment.
Purpose of the Study:
- To quantify the extent of off-label drug use in hospitalized children in the United States.
- To identify specific drugs frequently prescribed off-label.
- To determine patient and institutional factors associated with off-label medication use.
Main Methods:
- Retrospective cohort study utilizing administrative data from 31 tertiary care pediatric hospitals.
- Analysis of inpatient resource utilization data from 2004 for patients aged 18 years or younger.
- Off-label use defined as administration of a drug outside its FDA-approved age range.
Main Results:
- Off-label drug use occurred in 78.7% of 355,409 pediatric inpatients, representing 40.5% of total medication costs.
- Central/autonomic nervous system agents, fluids/nutrients, and gastrointestinal agents were most commonly used off-label.
- Factors associated with off-label use included surgical procedures, age >28 days, greater illness severity, and in-hospital mortality.
Conclusions:
- A significant majority of hospitalized children receive medications off-label, highlighting a critical area in pediatric pharmacotherapy.
- Variations in off-label use frequency were noted across different diagnostic groups and drug classes.
- While this study identifies frequent off-label use, it cannot ascertain the safety, efficacy, or clinical benefit of these treatments.
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.
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