{"title":"Off-label drug in the newborn","authors":"L. Cuzzolin","doi":"10.7363/030224","DOIUrl":null,"url":null,"abstract":"The lack of specific drugs and labelling recommendations for the neonatal population is a long-standing problem throughout the world. With the introduction of the Paediatric Regulation in 2007, in Europe tangible steps have been made to increase clinical research in children, but only a limited number of clinical trials included neonates that remain therapeutic orphans. This leads to a widespread use of medicines outside the terms indicated in the product license (off-label as regards dose, route of administration, indication, age group) or in an unlicensed manner (formulations modified, extemporaneous preparations, imported medicines, chemicals used as drugs). This use, often made on the basis of a consolidated clinical experience in absence of other authorized options, does not imply that a drug is contraindicated or disapproved, but simply means that insufficient data are available to grant approval status and the risks and benefits of using a drug in a particular situation have not been examined. Given the importance that neonatal population not be denied of drugs that are clearly beneficial, an updated overview of the worldwide situation of off-label and unlicensed drug use in the newborn will be presented, by analyzing also the impact of recent legislative initiatives and the well recognized problems (increased risk of ineffective or toxic treatments, adverse drug reactions and medication errors). Proceedings of the 10 th International Workshop on Neonatology · Cagliari (Italy) · October 22 nd -25 th , 2014 · The last ten years, the next ten years in Neonatology Guest Editors: Vassilios Fanos, Michele Mussap, Gavino Faa, Apostolos Papageorgiou","PeriodicalId":51914,"journal":{"name":"Journal of Pediatric and Neonatal Individualized Medicine","volume":"3 1","pages":""},"PeriodicalIF":0.3000,"publicationDate":"2014-10-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"10","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Pediatric and Neonatal Individualized Medicine","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.7363/030224","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 10
Abstract
The lack of specific drugs and labelling recommendations for the neonatal population is a long-standing problem throughout the world. With the introduction of the Paediatric Regulation in 2007, in Europe tangible steps have been made to increase clinical research in children, but only a limited number of clinical trials included neonates that remain therapeutic orphans. This leads to a widespread use of medicines outside the terms indicated in the product license (off-label as regards dose, route of administration, indication, age group) or in an unlicensed manner (formulations modified, extemporaneous preparations, imported medicines, chemicals used as drugs). This use, often made on the basis of a consolidated clinical experience in absence of other authorized options, does not imply that a drug is contraindicated or disapproved, but simply means that insufficient data are available to grant approval status and the risks and benefits of using a drug in a particular situation have not been examined. Given the importance that neonatal population not be denied of drugs that are clearly beneficial, an updated overview of the worldwide situation of off-label and unlicensed drug use in the newborn will be presented, by analyzing also the impact of recent legislative initiatives and the well recognized problems (increased risk of ineffective or toxic treatments, adverse drug reactions and medication errors). Proceedings of the 10 th International Workshop on Neonatology · Cagliari (Italy) · October 22 nd -25 th , 2014 · The last ten years, the next ten years in Neonatology Guest Editors: Vassilios Fanos, Michele Mussap, Gavino Faa, Apostolos Papageorgiou
期刊介绍:
The Journal of Pediatric and Neonatal Individualized Medicine (JPNIM) is a peer-reviewed interdisciplinary journal which provides a forum on new perspectives in pediatric and neonatal medicine. The aim is to discuss and to bring readers up to date on the latest in research and clinical pediatrics and neonatology. Special emphasis is on developmental origin of health and disease or perinatal programming and on the so-called ‘-omic’ sciences. Systems medicine blazes a revolutionary trail from reductionist to holistic medicine, from descriptive medicine to predictive medicine, from an epidemiological perspective to a personalized approach. The journal will be relevance to clinicians and researchers concerned with personalized care for the newborn and child. Also medical humanities will be considered in a tailored way. Article submission (original research, review papers, invited editorials and clinical cases) will be considered in the following fields: fetal medicine, perinatology, neonatology, pediatrics, developmental programming, psychology and medical humanities.