New paediatric research initiatives in the European Union

Abstract

In contrast to the situation concerning adults, most medicines used to treat the children of Europe have not been tested on children and are not authorised for use in children. Therefore, the health and quality of life of children in the EU countries may suffer from a lack of testing and authorisation of medicines for their use. In particular, 46% of medicines prescribed to children in hospital are either unlicensed for their age group or, if they are, have been done so off-label [1]. Of the children who receive medication in hospital this figure rises to 67% [1], and in the context of intensive care up to 90% of paediatric medicines used are not licensed [2]. Although there may be concerns voiced about conducting trials in the paediatric population, this has to be balanced by the ethical concerns related to giving medicines to a population in which they have not been tested and therefore their effects, positive or negative, are unknown. The European Parliament and Council Regulation on Medicinal Products for Paediatric Use [3] aims to improve the health of the children of Europe by increasing the research, development and authorisation of medicines for use in children, and as such represents a major breakthrough in paediatrics research. Its policy objectives are: to increase the development of medicines for use in children; to ensure that medicines used to treat children are subject to high quality research; to ensure that medicines used to treat children are appropriately authorised for use in children; to improve the information available on the use of medicines in children; and to achieve the above while avoiding unnecessary studies in children. Ensuring that children have access to high-quality, effective and safe medicines, accompanied by high-quality information based on robust evidence, is crucial to giving children and their doctors the ability to make informed decisions about the treatment of disease and ensuring that the chosen medicines improve health. However, the diseases suffered by children often differ considerably from those suffered by adults, and the bodies of children tolerate drugs differently from those of adults. The dose of a medicine to treat a childhood disease cannot always be extrapolated from the adult dose: medicinal products used in the paediatric population have never been specifically studied or authorised (licensed) for use in that age group. This leaves no alternative for the prescriber than to use products off-label – i.e. the use