Guide to Paediatric Drug Development and Clinical Research

Abstract

This series of monographs covers the latest in paediatric drug development and areas of clinical research. It also gives the reader a background to the enormous strides being made globally by agencies such as the European Medicines Agency, and the impact that the Food and Drugs Administration (FDA) Modernisation Act exclusivity incentive have had in encouraging paediatric drug research and development. As the introduction to this book suggests, children in the developed world have never had it so good as far as modern medical care and longevity are concerned. It is noteworthy that more than half of babies born in any G8 country today will live to 100 years based on current life expectancy trends. However (and embarrassingly so for the developed world), the monograph examining global access to essential medicines for children reminds us that although children in the developing world are increasingly enjoying the fruits of better healthcare, the morbidity and mortality rates for treatable medical conditions remain disturbingly high. The WHO Better Medicines for Children programme is clearly committed to narrowing this ever increasing global disparity in children's health and welfare, nevertheless the challenges faced by this organization will mean that at least one third of the world's population will remain ‘therapeutic orphans.’ The use of unlicensed and ‘off label’ medicines in children is widespread and yet drug toxicity remains a significant issue. The majority of drugs prescribed for children have not been adequately or properly tested in paediatric populations and without sufficient information and adequate data arising from further studies, children will remain the poor relations to their adult counterparts in the area of paediatric drug research and development. It is estimated that one in 10 children in hospital are likely to experience an adverse drug reaction. To address this and other concerns, governments, European regulation and the pharmaceutical industry are all applying a concerted approach in the area of paediatric drug development and clinical research, and there are several useful monographs in this book dealing with the regulatory and practical aspects of such developments. No guide to paediatric research can ignore the ethical and practical issues that have to be addressed when embarking on a paediatric pharmacological study and the book's monograph on this topic deals with these issues both adequately and succinctly. The last few chapters of this book deal with the practicalities of drug research trials in paediatric infections and vaccinations, paediatric obesity, hypertension and familial dyslipidaemias and attempt to deal with the thorny issue of using placebo therapy in children enrolled in randomized controlled trials. In conclusion, the editors of this book have brought together a diverse array of opinion in paediatric drug development. Any individual or organization embarking upon clinical research and paediatric drug development would be well placed in having read and considered some of the chapters in this book which identify the ethical, economic, logistical and technical issues currently hindering paediatric drug development. The ultimate expectation from carefully considered and conducted clinical trials is that children worldwide will benefit from the strides being made in this important area of children's health.