Attivita regolatoria europea ed uso 'off-label' dei farmaci in eta pediatrica

The purpose of licensing is to control the manufacture, provision, promotion and supply of medicines. To fulfil that purpose medicines are examined by regulatory agencies for safety, efficacy and quality. Many new medicines and the vast majority of older medications have been approved without labelling for paediatric use because research for establishing their safety and efficacy in children has not been carried out. The new European guidance on clinical investigation of medicinal products in children states that children should not be given medicines which have not been evaluated for use in that age group. The European legislation, however, makes provision for physicians to use medicines that do not have a marketing authorisation (unlicensed) and for purposes other than those stated in the marketing authorisation (off-label). Consequently, many medicines are prescribed for children without specific knowledge of the dosage, metabolism and potential side effects. The absence of appropriate usage information of the product in paediatric patients prevents health professionals to administer the drug in a manner that maximises safety and optimises treatment efficacy. This longstanding underprivileged position of children in respect of medicines could be improved by the new European drug registration system supported by the European Medicines Evaluation Agency. This paper evaluates the current status with regard to labelling for paediatric use of new medicines granted a pan-European marketing authorisation. It also describes current initiatives in Europe to facilitate and encourage drug research in children in order to achieve the necessary drug labelling to reduce unapproved uses of medication in children.