Inadequate pharmacotherapeutic data for drugs used in children: what can be done?

Although infants and children are affected by many acute and chronic diseases, nearly 80% of the drugs approved in the US for use in adults have not been labelled for paediatric use. This leads to the 'off-label' use of drugs which may produce suboptimal efficacy or harmful effects. Recent regulations proposed by the US Food and Drug Administration (FDA) require the industry to conduct paediatric studies in certain situations. However, some incentives will be provided to the industry to seek paediatric labelling. Paediatric practitioners and researchers will play an active role in conducting research and disseminating data about medication use in infants and children. However, increased funding is required for paediatric research. Industry, government, associations, academia and the public should work together to develop an agenda for action, with the goal of improving clinical outcomes and quality of life in infants and children.