Enabling Development of Paediatric Medicines in Europe: 10 Years of the EU Paediatric Regulation
- 325 Downloads
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.
Compliance with Ethical Standards
Conflicts of interest
Paolo A. Tomasi, Gunter F. Egger, Chrissi Pallidis and Agnes Saint-Raymond are all full-time employees of the EMA and have no conflicts of interest.
No funding was received for the conduct of this study and the preparation of the manuscript by any of the authors.
The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the European Medicines Agency or one of its committees or working parties.
- 3.Evidence of harm from off-label or unlicensed medicines in children. London: European Medicines Agency; 2004. http://www.ema.europa.eu/docs/en_GB/document_library/Other/2009/10/WC500004021.pdf. Accessed 16 Mar 2017.
- 6.European Commission. Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal medicines for pediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation (EC) No 726/2004. http://ec.europa.eu/health/files/eudralex/vol-1/reg_2006_1901/reg_2006_1901_en.pdf. Accessed 16 Mar 2017.
- 8.EMA Decision P/0065/2014. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/pips/EMEA-001462-PIP01-13/pip_001156.jsp&mid=WC0b01ac058001d129. Accessed 16 Mar 2017.
- 9.EMA Decision P/0291/2015. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/pips/EMEA-001820-PIP01-15/pip_001397.jsp&mid=WC0b01ac058001d129. Accessed 16 Mar 2017.
- 10.Annex—10-year Report to the European Commission. 2016. https://ec.europa.eu/health/sites/health/files/files/paediatrics/2016_pc_report_2017/ema_10_year_report_annex.pdfAccessed 17 Mar 2017.
- 12.Ruperto N, Vesely R, Saint-Raymond A, Martini A, Pediatric Rheumatology International Trials Organisation (PRINTO). Impact of the European pediatric legislation in pediatric rheumatology: past, present and future. Ann Rheum Dis. 2013;72(12):1893–6. doi: 10.1136/annrheumdis-2013-204168.CrossRefPubMedGoogle Scholar
- 14.European network of paediatric research at the European Medicines Agency (Enpr-EMA). London: EMA; 2017. http://www.ema.europa.eu/ema/index.jsp?curl=pages/partners_and_networks/general/general_content_000303.jsp&mid=WC0b01ac05801df74a . Accessed 15 July 2017.
- 16.EnprEMA database, 2017. http://enprema.ema.europa.eu/enprema/showall.php. Accessed 16 Mar 2017.
- 17.Lepola P, et al. Pharmaceutical industry and pediatric clinical trial networks in Europe—how do they communicate? Applied Clinical Trials online. 2016. http://www.appliedclinicaltrialsonline.com/pharmaceutical-industry-and-pediatric-clinical-trial-networks-europe-how-do-they-communicate. Accessed 11 May 2017.
- 18.Pediatric medicines: workshops. London: EMA; 2017. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000416.jsp&mid=WC0b01ac0580925cc6. Accessed 16 Mar 2017.
- 21.The 2017 Commission Report on the Paediatric Regulation. https://ec.europa.eu/health/human-use/paediatric-medicines/developments/2016_pc_report_2017_en. Accessed 11 May 2017.
- 23.European Medicines Agency. EU Clinical Trials Register. https://www.clinicaltrialsregister.eu/ctr-search/search. Accessed 23 Aug 2017.
- 24.10-year Report to the European Commission—General report on the experience acquired as a result of the application of the Paediatric Regulation. London: European Medicines Agency; 2016. https://ec.europa.eu/health/sites/health/files/files/paediatrics/2016_pc_report_2017/ema_10_year_report_for_consultation.pdf. Accessed 10 June 2017.
- 25.Zoely - Summary of Product Characteristics / EPAR. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/001213/WC500115831.pdf. Accessed 23 Aug 2017.
- 26.Results report of Lixisenatide study 2011-004584-67. https://www.clinicaltrialsregister.eu/ctr-search/trial/2011-004584-67/results Accessed 23 Aug 2017.
- 27.The 2017 Commission Report on the Paediatric Regulation. EC 2016: https://ec.europa.eu/health/sites/health/files/files/paediatrics/2016_pc_report_2017/ema_10_year_report_for_consultation.pdf. Accessed 23 Aug 2017.
- 29.European Parliament resolution of 15 December 2016 on the regulation on pediatric medicines. 2016. http://www.europarl.europa.eu/sides/getDoc.do?type=TA&language=EN&reference=P8-TA-2016-0511. Accessed 17 Mar 2017.
- 30.AAP makes recommendations on use of off-label drugs for children. Press release; 24 February 2014. https://www.aap.org/en-us/about-the-aap/aap-press-room/pages/AAP-Makes-Recommendations-On-Use-of-Off-Label-Drugs-for-Children.aspx. Accessed 4 May 2017.