title: Comparative Assessment of Off-label and Unlicensed Drug Prescriptions in Children: FDA Versus ANSM Guidelines.
abstract: The main objectives of this study were to assess the incidence of off-label (OL) and/or unlicensed (UL) prescriptions in a sample of pediatric Lebanese patients by using US Food and Drug Administration (FDA) and the French Medical Regulatory Authority (ANSM) regulations. The goal was to analyze the divergences between regulations and to identify those drugs most commonly involved in OL-UL utilization.
  
  
  
  This study was a retrospective analysis (500 pediatric files) conducted in a Lebanese University hospital in 3 pediatric wards (chronic diseases, acute diseases, and the pediatric intensive care unit).
  
  
  
  The frequency of OL-UL drug use was significantly different between pediatric wards (P < 0.001), with the highest incidence occurring in the intensive care unit. The most frequent OL-UL prescriptions occurred with cancer (oncology) admissions. Age was significantly related to OL-UL frequency (highest incidence in children aged between 0 and 1 year). The number of drugs prescribed per patient ranged between 1 and 20 (mean [SD], 4.13 [2.6]). The incidence of OL-UL prescriptions was significantly higher in patients treated with a greater number of medicines (P < 0.001). Overall, 58.9% of drug prescriptions were authorized according to ANSM and 50.7% according to FDA regulations; 11.1% (ANSM) and 15.8% (FDA) were UL, and 30.2% (ANSM) and 33.5% (FDA), respectively, were OL use (where OL for the indication were the most common). The highest percentage of OL-UL prescriptions was seen with the following groups: blood and blood-forming organs, genitourinary system, and sex hormones. Divergence between FDA and ANSM was mainly observed for OL medicines. UL prescriptions assessed according to both regulations showed similar results.
  
  
  
  This study highlights the need for prescribers to continuously examine updates to official regulations to avoid using an OL-UL drug whenever possible. It also calls for better harmonization between worldwide official guidelines concerning drugs used in children to reduce risk factors for adverse drug reactions.
authors.affiliation: Laboratoire de pharmacologie, pharmacie clinique et contrôle de qualité des médicaments, Faculty of Pharmacy, Saint-Joseph University of Beirut, Beirut, Lebanon.
authors.forename: Sandra
authors.initials: S
authors.lastname: Berdkan
authors.affiliation: Pediatric Department, Tennova Medical Center, Knoxville, Tennessee.
authors.forename: Lara
authors.initials: L
authors.lastname: Rabbaa
authors.affiliation: Laboratoire de pharmacologie, pharmacie clinique et contrôle de qualité des médicaments, Faculty of Pharmacy, Saint-Joseph University of Beirut, Beirut, Lebanon.
authors.forename: Aline
authors.initials: A
authors.lastname: Hajj
authors.affiliation: Pediatric Department, Hôtel-Dieu de France Hospital, Saint-Joseph University of Beirut, Beirut, Lebanon; Faculty of Medicine, Saint-Joseph University of Beirut, Beirut, Lebanon.
authors.forename: Bassam
authors.initials: B
authors.lastname: Eid
authors.affiliation: Faculty of Medicine, Saint-Joseph University of Beirut, Beirut, Lebanon; Department of Anesthesia and Critical Care, Hôtel-Dieu de France Hospital, Saint-Joseph University of Beirut, Beirut, Lebanon.
authors.forename: Hicham
authors.initials: H
authors.lastname: Jabbour
authors.affiliation: Department of Public Health, Faculty of Medicine, Saint-Joseph University, Beirut, Lebanon; Department of Prosthodontics, Faculty of Dental Medicine, Saint-Joseph University, Beirut, Lebanon.
authors.forename: Nada El
authors.initials: NE
authors.lastname: Osta
authors.affiliation: Laboratoire de pharmacologie, pharmacie clinique et contrôle de qualité des médicaments, Faculty of Pharmacy, Saint-Joseph University of Beirut, Beirut, Lebanon; Pharmacy Department, Hôtel-Dieu de France Hospital, Saint-Joseph University of Beirut, Beirut, Lebanon.
authors.forename: Latife
authors.initials: L
authors.lastname: Karam
authors.affiliation: Laboratoire de pharmacologie, pharmacie clinique et contrôle de qualité des médicaments, Faculty of Pharmacy, Saint-Joseph University of Beirut, Beirut, Lebanon. Electronic address: lydia.khabbaz@usj.edu.lb.
authors.forename: Lydia Rabbaa
authors.initials: LR
authors.lastname: Khabbaz
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D011307:Drug Prescriptions / Q000706:statistics & numerical data*; D064420:Drug-Related Side Effects and Adverse Reactions; D005260:Female; D006760:Hospitalization; D006785:Hospitals, University; D006801:Humans; D015994:Incidence; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D056687:Off-Label Use; D010818:Practice Patterns, Physicians' / Q000706:statistics & numerical data*; D012189:Retrospective Studies; D012307:Risk Factors; D014481:United States; D014486:United States Food and Drug Administration
keywords: ANSM; FDA; hospital; off-label; pediatric prescriptions; unlicensed