Special situations III: Medicines for Children

  • Christoph Male


The majority of drugs have never been evaluated for use in children. Developmental differences between adults and children of different ages affect pharmacokinetics and pharmacodynamics, and the safety profile of drugs. Use of drugs without paediatric information carries risks such as inappropriate dosing, lack of efficacy, and unexpected adverse events. Paediatric drug studies have been hampered by ethical and legal restrictions, methodological challenges and economical restraints. Recently, regulatory initiatives to stimulate paediatric drug development have been implemented in the US and EU. The EU Paediatric Regulation “Better Medicines for Children” requires paediatric development according to a Paediatric Investigation Plan (PIP) for all new drugs and on-patent drugs when applying for an authorization extension. Paediatric development is rewarded with a 6-months patent extension. PIPs are reviewed and amended by a Paediatric Committee at the European Medicines Agency. Certain collateral measures are included that are intended to improve information, transparency, and to stimulate research into paediatric medicines. Key points to consider for a PIP such as the definition of relevant paediatric indications(s), development of age- appropriate formulation(s), juvenile animal studies, paediatric PK and PD studies, and clinical efficacy and safely studies are discussed. A case study on a PIP is provided.


Developmental pharmacological differences EU Paediatric Regulation Paediatric Investigation Plan inventory of paediatric therapeutic needs age-appropriate formulation juvenile animal studies physiologically-based PK model population PK extrapolation 


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© Springer-Verlag/Wien 2010

Authors and Affiliations

  • Christoph Male
    • 1
  1. 1.Department of PaediatricsMedical University of ViennaViennaAustria

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