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Moving toward a paradigm shift in the regulatory requirements for pediatric medicines


Over the past two decades, there has been growing concern over the lack of proper medication for children. This review attempts to evaluate the current progress of EU Pediatric Regulation made since 2007. The lack of properly evaluated pediatric medication has for long been a source of concern in the European Union. The drugs that were used in the past were often not properly evaluated, and dosage was arbitrarily calculated. Therefore, it was necessary to establish the Pediatric Regulation (EC no. 1901/2006) in the EU which would mandate research for pediatric drugs. Current legislations in place not only require mandatory research by pharma industry but also have guidelines to direct the quality of pediatric research performed. The main aim of this regulation was to advance high-quality research and development of pediatric drugs, thereby increasing the availability of safe and effective drugs for children. It also aimed to improve the information available on existing pediatric drugs. It has been 9 years since the pediatric regulation was framed. The pharma industry now sees pediatric research as an integral process of development. Drug companies which develop plans for a new drug, new form of drug, new indication, or new route of administration for adults are obliged to integrate in their development plan similar research for pediatric populations as well.

Conclusion: It is hoped that the implementation of the current legislation will be reflected better in the future by the marketing of better and safer drugs for the pediatric population. The upcoming assessment to the European Commission in 2017 will further inform us on the impact after 10 years implementation of the legislation.

What is Known:
The lack of properly evaluated pediatric medication has for long been a source of concern in the European Union.
Therefore, it was necessary to establish the EU Pediatric Regulation which would mandate research for pediatric drugs.
What is New:
It has been 9 years since the pediatric regulation was framed, and the teething problems are slowly being overcome and the regulation is being used with increasing confidence.
As the Regulation is due for revision in 2017, this paper gives a current perspective on the impact of the regulation on availability and access to medicine for children.

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    EMA annual reports are available at http://www.ema.europa.eu/ema/index.jsp?curl=pages/about_us/document_listing/document_listing_000208.jsp&mid=WC0b01ac058002933a)



Best Pharmaceuticals for Children Act


European Pediatric Formulation Initiative


European Union


European Medicines Agency


European Network of Pediatric Research at the European Medicines Agency


European Network for Cancer Research in Children and Adolescents


Food and Drug Administration


Food and Drug Administration Modernization Act


Food and Drug Administration Safety and Innovation Act


Global Research in Pediatrics–Network of Excellence


International Conference on Harmonization


International Children’s Advisory Network


Pediatric Committee


Pediatric Investigation Plan


Pediatric Research Equity Act


Research and Development


Relating Expectations and Needs to the Participation and Empowerment of Children in Clinical Trials


Safety and Toxicity of Excipient Toxicity


United States


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Authors’ contributions

WWL. Chin collected data and figures and drafted the manuscript. A. Joos commented and revised the final manuscript.

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Correspondence to William Wei Lim Chin.

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Conflict of interest

WWL. Chin is an employee at the Hannover Medical School. A. Joos is an employee of MSD (Europe) Inc. The opinions expressed in this article are those of the authors and do not necessarily reflect the views of their employers or organizations. Both authors declare that they have no conflicts of interest in the research.

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This article does not contain any studies with human participants or animals performed by any of the authors.

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Communicated by Mario Bianchetti

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Chin, W.W.L., Joos, A. Moving toward a paradigm shift in the regulatory requirements for pediatric medicines. Eur J Pediatr 175, 1881–1891 (2016). https://doi.org/10.1007/s00431-016-2781-z

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  • Clinical trials
  • European regulation
  • Pediatric medicines
  • Pharmaceutical development
  • Regulatory framework