The EU paediatric regulation: still a large discrepancy between therapeutic needs and approved paediatric investigation plans
Wimmer S, Rascher W, Mccarthy S, Neubert A (2014)
Publication Type: Journal article
Publication year: 2014
Journal
Publisher: Adis International Ltd.
Book Volume: 16
Pages Range: 397-406
Journal Issue: 5
DOI: 10.1007/s40272-014-0082-4
Abstract
Prior to the implementation of the EU Paediatric Regulation, the European Medicines Agency (EMA) defined unmet paediatric needs for active substances already available on the market. Seven years after the Paediatric Regulation came into force, we investigated the extent to which previously identified needs have led to programmes for generating evidence necessary for the regulatory approval of medicines for managing childhood conditions.The websites of the EMA and the European Commission Community Research and Development Information Service (CORDIS) were systematically screened to identify active substances from the assessment of paediatric needs, off-patent priority list, agreed Paediatric Investigation Plans (PIP) and 7th Framework Programme (FP7) projects related to paediatric medicines.A total of 357 active substances with paediatric needs were identified by June 2013. 511 PIPs were agreed by the Paediatric Committee at the EMA (PDCO), including 51 (14.3 %) PIPs for a previously identified need. Amongst those, 21 were off-patent at the time of the PIP approval, 15 of which received funding from the European Commission's FP7. According to the assessment of paediatric needs, evidence is particularly needed for active substances treating cardiovascular diseases (n = 61), cancer (n = 40) and in the field of anaesthesiology (n = 38). Whereas oncology drugs (n = 66) were frequently represented in PIPs, drugs for cardiovascular diseases (n = 39) and anaesthesiology (n = 3) rarely were.Most PIPs are attributable to marketing authorisations of new active substances, whereas off-patent drugs which are commonly used off-label remain unstudied to a large extent. More effort including ongoing research funding is essential to further regularise and standardise paediatric pharmacotherapy.
Authors with CRIS profile
Wolfgang Rascher
Lehrstuhl für Kinder- und Jugendmedizin
Antje Neubert
Department of Paediatrics and Adolescent Medicine
Involved external institutions
Ireland (IE)How to cite
APA:
Wimmer, S., Rascher, W., Mccarthy, S., & Neubert, A. (2014). The EU paediatric regulation: still a large discrepancy between therapeutic needs and approved paediatric investigation plans. Paediatric Drugs, 16(5), 397-406. https://doi.org/10.1007/s40272-014-0082-4
MLA:
Wimmer, Stefan, et al. "The EU paediatric regulation: still a large discrepancy between therapeutic needs and approved paediatric investigation plans." Paediatric Drugs 16.5 (2014): 397-406.
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