Toma Maddalena, Felisi Mariagrazia, Bonifazi Donato, Bonifazi Fedele, Giannuzzi Viviana, Reggiardo Giorgio, de Wildt Saskia, Ceci Adriana
Fondazione per la Ricerca Farmacologica "Gianni Benzi" Onlus, Bari, Italy.
Consorzio per Valutazioni Biologiche e Farmacologiche, Bari, Italy.
Front Med (Lausanne). 2021 Feb 2;8:593281. doi: 10.3389/fmed.2021.593281. eCollection 2021.
In this paper, we investigated the effects of the European Paediatric Regulation (EC) N° 1901/2006 with respect to satisfying the paediatric therapeutic needs, assessed in terms of the increased number of paediatric medicinal products, new therapeutic indications in specific high-need conditions (neonates, oncology, rare disease, etc.) and increased number of paediatric clinical studies supporting the marketing authorisation. We analysed the paediatric medicinal products approved by the European Medicines Agency in the period January 2007-December 2019, by collecting the following data: year of approval, active substance, legal basis for the marketing authorisation, type of medicinal product (i.e., chemical, biological, or ATMP), orphan drug status, paediatric indication, Anatomical Therapeutic Chemical code (first-level), number and type of paediatric studies. Data were compared with similar data collected in the period 1996-2006. In the period January 1996-December 2019, in a total of 1,190 medicinal products and 843 active substances, 34 and 38%, respectively, were paediatric. In the two periods, before and after the Paediatric Regulation implementation, the paediatric/total medicinal products ratio was constant while the paediatric/total active substances ratio decreased. Moreover, excluding generics and biosimilars, a total of 106 and were granted a in the two periods; out of 175, 128 paediatric medicines had an approved Paediatric Investigational Plan. The remaining 47 were approved without an approved Paediatric Investigational Plan, following the provisions of Directive 2001/83/EC and repurposing an drug. The analysis of the clinical studies revealed that drugs with a Paediatric Investigational Plan were supported by 3.5 studies/drug while drugs without a Paediatric Investigational Plan were supported by only 1.6 studies/drug. This report confirms that the expectations of the European Paediatric Regulation (EC) N° 1901/2006 have been mainly satisfied. However, the reasons for the limited development of paediatric medicines in Europe, should be further discussed, taking advantage of recent initiatives in the regulatory field, such as the Action Plan on Paediatrics, and the open consultation on EU Pharmaceutical Strategy.
在本文中，我们调查了欧盟儿科法规（EC）第1901/2006号在满足儿科治疗需求方面的影响，这些影响通过儿科药品数量的增加、特定高需求病症（新生儿、肿瘤学、罕见病等）的新治疗适应症以及支持上市许可的儿科临床研究数量的增加来评估。我们分析了欧洲药品管理局在2007年1月至2019年12月期间批准的儿科药品，收集了以下数据：批准年份、活性物质、上市许可的法律依据、药品类型（即化学药、生物药或先进治疗药物产品）、孤儿药状态、儿科适应症、解剖治疗化学代码（一级）、儿科研究的数量和类型。将这些数据与1996年至2006年期间收集的类似数据进行了比较。在1996年1月至2019年12月期间，总共1190种药品和843种活性物质中，儿科药品和活性物质分别占34%和38%。在儿科法规实施前后的两个时期，儿科药品与总药品的比例保持不变，而儿科活性物质与总活性物质的比例下降。此外，排除仿制药和生物类似药后，两个时期分别有106种和……被授予了……；在175种儿科药品中，128种有批准的儿科研究计划。其余47种是根据2001/83/EC指令的规定，在没有批准的儿科研究计划的情况下，通过重新利用一种……药物而获得批准的。对临床研究的分析表明，有儿科研究计划的药物平均每种有3.5项研究支持，而没有儿科研究计划的药物平均每种只有1.6项研究支持。本报告证实，欧盟儿科法规（EC）第1901/2006号的期望已基本得到满足。然而，应利用监管领域的近期举措，如儿科行动计划和欧盟制药战略公开咨询，进一步讨论欧洲儿科药品开发受限的原因。
