Department of Clinical Pharmacology, Bispebjerg Hospital, 2400, Copenhagen, Denmark.
Eur J Clin Pharmacol. 2014 Apr;70(4):445-52. doi: 10.1007/s00228-013-1626-1. Epub 2014 Jan 8.
This nationwide study is aimed at describing to what extent the European Paediatric Regulation has met therapeutic needs in children.
Data for each drug substance in defined daily doses (DDD) were extracted from the national Danish data base. We evaluated if drug substances were used off-label and whether they had a paediatric investigation plan (PIP). This study did not include drug prescriptions for individual paediatric patients; thus, it was not possible to make use of all off-label categories previously used. Additionally, paediatric standard assortments (SA) were compared to the European survey on paediatric medicinal products.
Thirteen percent of the 100 most used drug substances were determined as being used off-label, four of which had a PIP and one had a full waiver. Only one of the three drug substances used off-label most often, accounting for 85 % of such use, had a PIP. Neonates were included in one-third of PIPs and adolescents in 15. Nineteen out of 21 PIPs had a waiver and 14 PIPs were deferred. In line with the European survey, carbapenems, corticosteroids and proton pump inhibitors were frequent found in SAs.
PIPs only cover a small proportion of the drugs found to be used off-label in this study. Despite waivers granted, drug substances were used nonetheless. Unmet regulatory needs are still considerable in some therapeutic areas in neonates as well as in children.
本全国性研究旨在描述《欧洲儿科法规》在多大程度上满足了儿童的治疗需求。
从丹麦国家数据库中提取每个按日剂量（DDD）定义的药物的药物数据。我们评估了药物是否被超适应证使用，以及它们是否有儿科研究计划（PIP）。本研究不包括针对个别儿科患者的药物处方；因此，无法利用以前使用的所有超适应证类别。此外，将儿科标准制剂（SA）与欧洲儿科药物调查进行了比较。
100 种最常用药物中有 13%被确定为超适应证使用，其中 4 种有 PIP，1 种有完全豁免。在最常被超适应证使用的 3 种药物中，只有 1 种有 PIP，占此类使用的 85%。新生儿在三分之一的 PIP 中被纳入，青少年在 15%的 PIP 中被纳入。21 个 PIP 中有 19 个获得豁免，14 个 PIP 被推迟。与欧洲调查一致，碳青霉烯类、皮质类固醇和质子泵抑制剂在 SA 中经常出现。
PIP 仅涵盖本研究中超适应证使用的药物的一小部分。尽管给予了豁免，但仍在使用药物。在某些治疗领域，新生儿和儿童仍存在相当大的监管需求未得到满足。
