An Overview of Off-Label Use of Humanized Monoclonal Antibodies in Paediatrics

Abstract

In recent years, off-label and unlicensed drug use has extensively developed in the paediatric population. For a long time, clinical trials in the paediatric population were considered complicated to perform because of ethical problems, causing frequent off-label use. Off-label drug use remains an important public health issue, especially for children with rare conditions or with diseases not responsive to conventional treatments. The present paper is a narrative review of the literature of off-label drug use in children. The aim of our study is to summarize the main works dealing with the off-label use of biological drugs in paediatrics. Further studies analyzing their efficacy, safety, and cost–benefit ratios are needed to extend the use of biological therapies to the paediatric population.

Pediatric Drug Safety Surveillance: A 10-Year Analysis of Adverse Drug Reaction Reporting Data in Calabria, Southern Italy

“…As a result, when prescribing medications to children, physicians frequently tend or have to draw their conclusions from adult drug trials, without age-specific information on correct dosing and/or on drug efficacy and safety [ 5 , 6 ]. Consistently, accruing evidence has shown a widespread use of unlicensed or ‘off-label’ drugs to treat children [ 7 – 10 ]. Off-label use reflects the prescription of drugs outside the terms of their marketing authorization with respect to dosage or frequency, age groups, clinical indication, or route; unlicensed use refers to: (1) licensed drugs whose formulation is modified; (2) drugs manufactured as extemporaneous preparations; (3) drugs imported or used before license granting; or (4) chemicals used as drugs for lack of pharmaceutical preparation [ 8 ].…”