Guide to Paediatric Clinical Research

Abstract

ROSE, KLAUS, AND VAN DEN ANKER, JOHN N.: GUIDE TO PAEDIATRIC CLINICAL RESEARCH. BASEL, SWITZERLAND: KARGER, 2007, 138 PAGES. The widespread use in pediatric patients of medicines licensed only for adults is a matter of growing concern to the profession, governments, regulatory agencies, and the public. It stands to reason that drugs should be studied in children to determine their safety and efficacy in younger age groups. All textbooks and reviews proudly state that growth, differentiation, and maturation can alter the kinetics, end organ responses, and toxicities to drugs in the newborn, infant, child, or adolescent compared with the adult. Drugs are subject to licensing procedures to ensure their safety, effectiveness, and quality. Running such studies in children often poses substantial ethical, practical, and commercial problems. Therefore, scientific data on the use of many pharmaceutical products in children are lacking. Such nonvalidated drug use has been challenged as a fundamental denial of the legal and human rights of children. Among experts from academia, regulatory agencies, and pharmaceutical companies, there is consensus that more studies are needed, so that in the future children can be treated with drugs that have been properly evaluated for use in their particular age group. Recently, US and EU governments have made it mandatory for the pharmaceutical industry to investigate medicines in children. This book contains 15 contributions written by 26 authors who are accepted experts in pediatric clinical research, from academia and industry. All chapters summarize the actual knowledge, emphasizing European situations and recommendations. The main topics include the relevant legislative changes and guidelines, the operational and ethical challenges of research involving children, the practical aspects related to informed consent/assent, appropriate drug formulation, and sample collection and analysis. Special chapters deal with research in small infants and with the complex issue of developing new infant nutrition products. This informative and welcome textbook is full of scientific and practical content. It will help to shift pediatric drug development from exception to routine. This book is recommended for colleagues with clinical, academic, pharmaceutical, or regulatory backgrounds who are interested and involved in clinical research with infants and children.