Belančić Andrej, Kučan Štiglić Marta, Rešić Arnes, Fajkić Almir, Vitezić Dinko
Department of Basic and Clinical Pharmacology and Toxicology, Faculty of Medicine, University of Rijeka, 51000 Rijeka, Croatia.
Children's Hospital Zagreb, 10000 Zagreb, Croatia.
J Clin Med. 2025 Aug 25;14(17):5994. doi: 10.3390/jcm14175994.
: Children remain underserved in pharmaceutical development, with off-label prescribing still prevalent in part due to a lack of age-appropriate formulations. This study aimed to evaluate the national uptake of Pediatric Use Marketing Authorisation (PUMA)-labelled medicines in Croatia from 2017 to 2024. : We conducted a retrospective, descriptive pharmacoepidemiological study using the IMS (Intercontinental Medical Statistics) and IQVIA (Information, Quintiles, VIA; formerly IMS Health and Quintiles) datasets to track utilization and the expenditure of all PUMA products. Utilization was assessed using defined daily doses per 1000 inhabitants per day (DDDs/1000/day) and annual product dispensation counts. : Over the study period, five PUMA medicines entered the Croatian market, with usage rising from 853 packages in 2018 to 9232 in 2024. The DDDs/1000/day increased 33.8-fold, while the expenditure escalated nearly 5.8-fold, from EUR 145,898 to EUR 844,145. Midazolam and melatonin were the most frequently prescribed, yet the overall utilization remained marginal relative to pediatric needs. : In conclusion, while regulatory availability of PUMA products has improved, their clinical adoption in Croatia remains limited. Addressing economic, educational, and policy barriers is essential to close the gap between authorization and utilization.
在药物研发方面，儿童仍然未得到充分服务，部分原因是缺乏适合儿童年龄的制剂，导致超说明书用药仍然普遍。本研究旨在评估2017年至2024年克罗地亚对带有儿科用途上市许可（PUMA）标签药品的全国使用情况。我们进行了一项回顾性描述性药物流行病学研究，使用IMS（洲际医学统计）和IQVIA（信息、昆泰、VIA；前身为IMS Health和昆泰）数据集来跟踪所有PUMA产品的使用情况和支出。使用每1000居民每天的限定日剂量（DDDs/1000/天）和年度产品配药计数来评估使用情况。在研究期间，五种PUMA药物进入克罗地亚市场，使用量从2018年的853包增加到2024年的9232包。DDDs/1000/天增加了33.8倍，而支出从145,898欧元增至844,145欧元，增长了近5.8倍。咪达唑仑和褪黑素是最常被处方的药物，但相对于儿科需求而言，总体使用量仍然很少。总之，虽然PUMA产品的监管可用性有所提高，但它们在克罗地亚的临床应用仍然有限。解决经济、教育和政策障碍对于缩小授权与使用之间的差距至关重要。
