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The EU Paediatric Regulation: Still a Large Discrepancy Between Therapeutic Needs and Approved Paediatric Investigation Plans

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Abstract

Purpose

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.

Methods

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.

Results

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.

Conclusions

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.

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Contributions of Authors Statement

AN and WR developed the idea and the design of the analysis. SW conducted data collection and analysis. SW and AN drafted the manuscript. WR and SM reviewed and revised the manuscript. AN is the guarantor of the manuscript.

Conflict of Interest

S. Wimmer, W. Rascher, S. McCarthy and A. Neubert declare no conflict of interest. No funding was received for this work.

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Authors and Affiliations

  1. Department of Paediatrics and Adolescent Medicine, University Hospital Erlangen, Loschgestraße 15, 91054, Erlangen, Germany

    Stefan Wimmer, Wolfgang Rascher & Antje Neubert

  2. School of Pharmacy, University College Cork, Cork, Ireland

    Suzanne McCarthy

Authors
  1. Stefan Wimmer
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  2. Wolfgang Rascher
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  3. Suzanne McCarthy
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  4. Antje Neubert
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Corresponding author

Correspondence to Antje Neubert.

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Wimmer, S., Rascher, W., McCarthy, S. et al. The EU Paediatric Regulation: Still a Large Discrepancy Between Therapeutic Needs and Approved Paediatric Investigation Plans. Pediatr Drugs 16, 397–406 (2014). https://doi.org/10.1007/s40272-014-0082-4

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