Paediatrics and Neonatology Unit, San Giuseppe Hospital, 50053 Empoli, Italy.
Specialty School of Paediatrics, Alma Mater Studiorum, University of Bologna, 40138 Bologna, Italy.
Medicina (Kaunas). 2022 Apr 29;58(5):625. doi: 10.3390/medicina58050625.
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.
近年来，标签外用药和无许可证用药在儿科人群中广泛发展。长期以来，由于伦理问题，儿科人群的临床试验被认为难以进行，导致频繁出现标签外用药。标签外用药仍然是一个重要的公共卫生问题，特别是对于患有罕见疾病或对常规治疗无反应的儿童。本文是一篇关于儿童标签外用药的文献综述。我们的研究目的是总结主要涉及儿科生物药物标签外使用的工作。需要进一步研究分析其疗效、安全性和成本效益比，以将生物治疗扩展到儿科人群。
