Enabling Development of Paediatric Medicines in Europe: 10 Years of the EU Paediatric Regulation
Enabling Development of Paediatric Medicines in Europe: 10 Years of the EU Paediatric Regulation
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DOI：
10.1007/s40272-017-0261-1
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发表时间：
2017-12-01
期刊：
影响因子：
3.7
通讯作者：
Saint-Raymond, Agnes
中科院分区：
文献类型：
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作者：
Tomasi, Paolo A.;Egger, Gunter F.;Saint-Raymond, Agnes
2017年是《儿科条例》在欧洲联盟(欧盟)生效十周年。这项法律旨在刺激儿科药物的开发，并提供更多关于其使用的信息，以应对儿童药物缺乏证据和缺乏批准的问题。欧洲药品管理局(EMA)在该条例的实施中发挥了核心作用。制药公司需要向EMA的儿科委员会(PDCO)提交每种新药的儿科调查计划(PIP)，除非获得豁免(豁免)。这些计划描述了儿童药物的开发，必须在申请药品上市授权之前很久就达成一致。可以批准推迟研究，以便在完成儿科研究之前批准成人进行研究。2007年1月至2016年12月，欧盟总共批准了273种适合儿童使用的新药和43种额外的药物形式，EMA同意了950种PIP。此外，还同意在一种或多种医疗条件下开发一种药物的486项豁免。《儿科条例》对儿科药物开发产生了非常积极的影响，对比《条例》生效前后的两个为期3年的时期就证明了这一点。我们的结论是，该条例导致了更多的儿童药物，并使临床医生能够获得更多关于欧盟儿科用药的信息。
The year 2017 marks the tenth anniversary of entry into force of the Paediatric Regulation in the European Union (EU). This law aimed to stimulate the development of paediatric medicines and provide more information on their use, as a response to the lack of evidence and approval of medicines for children. The European Medicines Agency (EMA) has had a central role in the implementation of the Regulation. Pharmaceutical companies need to submit a paediatric investigation plan (PIP) to the EMA's Paediatric Committee (PDCO) for every new medicine, unless an exemption (waiver) is granted. The plans, which describe the development of drugs for children, must be agreed well in advance of the request for marketing authorization of the medicine. Deferrals of studies can be granted to allow approval in adults before the completion of paediatric studies. Between January 2007 and December 2016, a total of 273 new medicines and 43 additional pharmaceutical forms appropriate for use in children were authorized in the EU, and 950 PIPs were agreed by the EMA. In addition, 486 waivers of the development of a medicine in one or more medical conditions were agreed. The Paediatric Regulation has had a very positive impact on paediatric drug development, as exemplified by a comparison of two periods of 3 years before and after entry into force of the Regulation. We conclude that the Regulation has resulted in more medicines for children and more information on the pediatric use of medicines in the EU being available to clinicians.