Special Situations III: Medicines for Children

Abstract

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 different adverse events as in adults. 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 USA 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 authorisation extension. Paediatric development is rewarded with a 6-month 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 and transparency and to stimulate research into paediatric medicines. Key points to consider for a PIP are the definition of relevant paediatric indications(s), development of age-appropriate formulation(s), juvenile animal studies, paediatric PK and PD studies, clinical efficacy and safety studies, and the possibility of extrapolation from adults. A case study on a PIP is provided.