Off-label and unlicensed prescribing for newborns and children in different settings: a review of the literature and a consideration about drug safety

Abstract

This review aims to give an updated overview of the worldwide situation of off-label and unlicensed drug use in the paediatric field, also taking into account the safety of this kind of treatment. A Medline and Embase search was performed between 1990 and 2006 and a total of 52 studies were identified and included in the systematic review. From the authors' analysis of the literature, the extent of paediatric unlicensed/off label use is higher in neonatal and paediatric intensive care units and oncology wards, compared with primary care. Moreover, among the nine studies reporting the contribution of an off-label/unlicensed drug use to the occurrence of adverse events, the percentage of unlicensed and/or off-label prescriptions involved in an adverse drug reaction ranged between 23 and 60%. To ensure that children are not exposed to unnecessary risks, controlled clinical trials are required. In addition, future research should be directed towards the identification of individual drugs that cause serious adverse drug reactions and lack product information.

Off-label drug use in pediatric anesthesia and intensive care according to official and pediatric reference formularies

“…3,9,14 It identifies the principal reasons why the most commonly prescribed off-label drugs were recognized as being off-label. The main objective for this study was to ascertain whether the frequency of off-label prescriptions would have been reduced substantially if an alternative method (e.g., contemporary pediatric references) were used to inform the official drug label.…”

“…Patients admitted to other units of the hospital were not considered as they have been reported elsewhere. 14 Data sources included the medical chart, the anesthetic record, the nursing medication administration record, and the pharmacy record. During the study month, a single reviewer recorded each drug prescription along with patient demographics (age, sex, weight, diagnosis or procedure undertaken).…”

Off-Label Prescribing in Pediatric Outpatients

“…However, administration of a drug outside the conditions assessed during clinical trials may result in adverse drug reactions (ADRs). [1][2][3] Moreover, the specificity of children's physiology and pharmacology should not be neglected. 4 For years, the lack of drug evaluation in children has been evoked as a factor favoring off-label prescribing.…”

Why do children decide not to participate in clinical research: a quantitative and qualitative study

“…M ore drug researches in children are needed, because currently 36-90% of drugs prescribed to children have not been tested in their age group (1). Prescribers often have no alternative but to resort to off-label or unauthorized products, without having evidence-based information regarding drug effects on children to guide them and help them weigh the risks and benefits of the drug.…”