Briefing Paper on Paediatric Medicines and Clinical Research

Lawrence D. Tobias was the primary author of a Briefing Paper on Paediatric Medicines and Clinical Research which was commissioned by the International Alliance of Patients' Organizations (IAPO) and released for distribution in November of 2006. The paper took nearly a year to complete and involved an intensive search of the extensive available literature on the topic; a thorough understanding of the varied international paediatric clinical trial-related regulations; and interviews with over one dozen leading international experts in the area of paediatric medicines, paediatric regulations, and clinical trial ethics. Quoting from the introduction to the paper: "The lack of medicines approved specifically for children is of global concern. In Europe, it is estimated that more than half of the medicines used to treat children are either untested or unauthorized for paediatric use (European Commission, 2004). It is essential that medicines be developed specifically for children, because the alternatives of either no medication available or administering off-label medicines expose children to unacceptable and unethical risks to their health."

IAPO states that "Our paper aims to equip patient advocates with an understanding of the specific issues relevant to paediatric medicines and clinical trials. It gives an overview of the regulatory, social, ethical and economic issues, as well as a summary of the existing and potential benefits and risks related to the issue, all based on current evidence-based research and expert opinions.

We hope that making this information accessible to patients’ organizations worldwide will enable them to make an informed judgement on the value of clinical trials for paediatric medicines. Actively engaging in debate and discussion with patients and other stakeholders in healthcare on this issue is crucial so that the patient voice is represented in new and developing legislation and medications. This is particularly timely as new legislation on the regulation of medicinal products for paediatric use in the European Union is due to come into force at the beginning of 2007."

Jo Harkness of IAPO was coauthor on the paper and contributed to the section dealing with the ethics of conducting clinical trials in children.

Information regarding purchase of the briefing paper can be found on the IAPO website at http://www.patientsorganizations.org/showarticle.pl?id=762.