Salvo Ida, Landoni Giovanni, Mucchetti Marta, Cabrini Luca, Pani Luca
Department of Anesthesia and Intensive Care, Children's Hospital V Buzzi, Milan, Italy.
Paediatr Anaesth. 2014 Jun;24(6):625-31. doi: 10.1111/pan.12403. Epub 2014 May 3.
Most of the drugs used in anesthesia are off-label in children even if they present solid clinical evidence in adults. This lack of authorization is caused by multiple factors including the difficulty in conducting research in this area (due to the ethical concerns and/or the low number of available participants, the high variability of the outcome measures) and the lack of economic interest of the pharmaceutical companies (due to the limited market).
Define a list of medicinal products commonly used off-label in pediatrics anesthesia to be reimbursed by Italian National Health System.
We hereby describe the methodological framework used to allow reimbursed use of a list of medicinal products, widely used off-label in pediatric patients, ensuring the best therapeutic results with the lowest possible risk for children. A task force of pediatric anesthesiologists from Italy petitioned the Italian Medicines Agency (AIFA) to allow a number of commonly utilized but off-label drugs for pediatric anesthesia to be reimbursed for specific indications. For each drug, both the supporting literature and expert opinion were used, and the resulting list of drugs allowed to be used/reimbursed officially by AIFA was significantly expanded. This paper documents one approach to the problem of off-label use of drugs for pediatric patients that can be a model for future efforts.
Continuous efforts are needed from government institutions and sponsors on drug development and on drug approval process in pediatrics, as research on drug effectiveness and safety is mandatory in children as in adults. At the same time, clinicians must become more familiar with the drug-approval process, participate to sponsored trials, and perform ztrials themselves.
麻醉中使用的大多数药物在儿童中属于超说明书用药，即便它们在成人中有确凿的临床证据。这种未获批准的情况是由多种因素造成的，包括在该领域开展研究存在困难（由于伦理问题和/或可用参与者数量少、结果测量的高度变异性）以及制药公司缺乏经济利益（由于市场有限）。
确定意大利国家卫生系统报销的儿科麻醉中常用的超说明书用药清单。
我们在此描述用于允许报销一系列在儿科患者中广泛超说明书使用的药品的方法框架，以确保在儿童风险尽可能低的情况下获得最佳治疗效果。来自意大利的儿科麻醉医生组成的一个特别工作组向意大利药品管理局（AIFA）请愿，请求允许一些儿科麻醉中常用但超说明书使用的药物用于特定适应症时可获得报销。对于每种药物，既使用了支持性文献，也参考了专家意见，AIFA正式允许使用/报销的药品清单得到了显著扩充。本文记录了一种解决儿科患者超说明书用药问题的方法，可作为未来努力的典范。
政府机构和赞助商需要在儿科药物研发和药物审批过程中持续努力，因为儿童药物有效性和安全性研究与成人一样必不可少。同时，临床医生必须更熟悉药物审批过程，参与赞助商发起的试验，并自行开展试验。
