Use and Safety of Respiratory Medicines in Children

The lack of appropriately authorised and formulated medicines for use in the pediatric population is a longstanding problem and cause for concern. As a result, most medicines are prescribed to children on an off -label or an unlicensed basis. Dosing regimens approved for adults are extrapolated to pediatric age groups, for example on the basis of proportionality of weight, without pediatric pharmacokinetic or pharmacodynamic data. Safety and effi cacy are presumed to be the same as in adults, but this may not be the case. Evidence-based information in children is often not readily available, and prescribing decisions may have to be based on accepted practice presented in formularies. On January 26, 2007, the new regulation of the European Union (EU) on medicinal products for pediatric use came into force. The overall aim of the regulation is to improve the health of children in the EU. More specifi cally the objectives include: increasing the development of medicines for use in children; ensuring that medicines used to treat children are subject to high quality research and appropriately authorised for use in children; improving the information available on the use of medicines in children; and achieving the above objectives without subjecting children to unnecessary clinical trials or delaying the authorisation of medicines in the adult population. Previously, pharmaceutical industry had been free to restrict a particular medicine’s development to the adult population; the development of pediatric medicines was solely at the industry’s discretion. The new regulation requires that all applications for marketing authorisation for new medicines, must contain the results of all studies and information required in a previously agreed pediatric investigation plan (PIP) The PIP will contain a full proposal of all the studies (and their timings) necessary to support the pediatric use of an individual product and will cover all pediatric age groups and all necessary age-appropriate formulations. In some case, pharmaceutical companies can obtain a deferral of waiver from a PIP, for example where there is no pediatric therapeutic need or where pediatric use of the product is not appropriate. Unless a deferral or a waiver has been granted, pediatric data must be provided in all applications for the authorisation of new medicinal products. The regulation also establishes a new type of marketing authorisation, called the pediatric use marketing authorization (PUMA), intended to stimulate the development of off -patent products for use in the pediatric population. Only medicines that are intended solely for use in children will be eligible for a PUMA. The new legislation obliges the European Medicines Agency to build one European network from the diff erent existing networks and collaborative projects, investigators, and specifi c Centers of Excellence.

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