Guide to Paediatric Drug Development and Clinical Research

Description

Children in the developed world have never enjoyed better medical care: mortality has decreased and many fatal diseases of the past can today be prevented or even cured. However, the current practice of pharmacotherapy in children does not reflect existing scientific knowledge and has come under scrutiny by paediatricians, pharmacists and regulatory authorities. In order to advance the development of medicines tailored to paediatric needs, US and EU legislators have taken action, and the WHO has initiated a global paediatric campaign. This book gives an overview over the worldwide activities that increasingly include children in the development of new medicines. Triggered by both a better understanding of how the child’s body develops as well as recent legislation in the USA and in Europe, this comprises dosing, ethics, age-appropriate pharmaceutical forms and clinical trials, to name just a few aspects. A wide spectrum of readers will profit from this book, including paediatricians, pharmacists, general practitioners and health care professionals involved in child care and paediatric research, clinical trial personnel, patient advocacy groups, ethics committees, politicians, parents and interested lay persons.

Informations

• Publié par: S. Karger AG
• Date de parution: 21 mai 2010
• Nombre de lectures: 0
• EAN13: 9783805593632
• Langue: English
• Poids de l'ouvrage: 1 Mo

Extrait

Guide to Paediatric Drug Development and Clinical Research
Guide to Paediatric Drug Development and Clinical Research
Editors
Klaus Rose Munich
John N. van den Anker Washington D.C./Rotterdam
19 figures, and 23 tables, 2010
_______________________ Klaus Rose Principal Consultant Granzer Regulatory Consulting & Services Zielstattstrasse 44 DE-81379 Munich, Germany
_______________________ John N. van den Anker Division of Pediatric Clinical Pharmacology Children's National Medical Center 111 Michigan Avenue, NW Washington, DC 20010, USA and Department of Pediatrics Erasmus MC – Sophia Children's Hospital Dr. Molewaterplein 60 NL-3015 GJ Rotterdam, The Netherlands
Library of Congress Cataloging-in-Publication Data
Guide to paediatric drug development and clinical research/editors, Klaus Rose, John N. van den Anker.
p.; cm.
Includes bibliographical references and indexes.
ISBN 978-3-8055-9362-5 (hard cover: alk. paper)
1. Pediatric pharmacology. 2. Drug development. I. Rose, Klaus, 1953- II. Van den Anker, John N.
[DNLM:1. Drug Evaluation. 2. Child. 3. Clinical Trials as Topic. 4. Infant. QV 771 G9463 2010]
RJ560.G85 2010
615'.190083––dc22
2010001239
Bibliographic Indices. This publication is listed in bibliographic services.
Disclaimer. The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publisher and the editor(s). The appearance of advertisements in the book is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements.
Drug Dosage. The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any change in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug.
All rights reserved. No part of this publication may be translated into other languages, reproduced or utilized in any form or by any means electronic or mechanical, including photocopying, recording, microcopying, or by any information storage and retrieval system, without permission in writing from the publisher.
© Copyright 2010 by S. Karger AG, P.O. Box, CH-4009 Basel (Switzerland)
www.karger.com
Printed in Switzerland on acid-free and non-aging paper (ISO 9706) by Reinhardt Druck, Basel
ISBN 978-3-8055-9362-5
e-ISBN 978-3-8055-9363-2
Contents
Introduction
The Editors
Authors’ Addresses
The Global Framework of Paediatric Drug Development
______________________
Europe and the Path to Better Medicines for Children
Ramet, J. (Edegem); Lehman, B. (Bonn); Rose, K. (Basel)
European Union Paediatric Regulation: Theory and Practice
Wright, E. (Brussels); Rose, K. (Basel)
United States Paediatric Legislation Impact on Paediatric Drug Studies
Rodriguez, W. (Rockville, Md.); Maldonado, S. (Raritan, N.J.)
Facilitation of Paediatric Research in Japan
Nakamura, H.; Ozaki, M. (Tokyo)
Ethical Imperatives in Medical Research on Child Subjects – Reflections of a Past President of the World Medical Association
Appleyard, W.J. (Kent)
Paediatric Clinical Research: The Patients ‘Perspective
Kent, A. (London/Brussels); Oosterwijk, C. (Brussels/Soest); Poortman, Y. (The Hague)
Providing Global Access to Essential Medicines for Children – The WHO Better Medicines for Children Programme
Hoppu, K. (Helsinki); Hill, S. (Geneva)
Paediatric Clinical Pharmacology
______________________
Paediatric Clinical Pharmacology: From Bench to Bedside
van den Anker, J.N. (Washington, D.C./Rotterdam)
Role of Clinical Pharmacology in the Development of Paediatric Clinical Development Plans
Reigner, B.; Ricci, B.M.; Liogier d'Ardhuy, X. (Basel)
Practical and Ethical Challenges
______________________
Guide through the EMA Paediatric Website
Rose, K. (Basel); Granzer, U. (Munich); Della Pasqua, O. (Stockley Park)
Paediatric Homework: Paediatric Investigation Plan Key Elements
Rose, K. (Basel); Della Pasqua, O. (Stockley Park)
Ethics in Paediatric Research: Three Years after the Introduction of the European Regulation
Matthys, D. (Ghent); Rose, K. (Basel)
Consent and Assent in Paediatric Clinical Trials
Simar, M.R. (Austin, Tex.); Fowler, P.A. (Hertford)
Study and Protocol Design for Paediatric Patients of Different Ages
Della Pasqua, O. (Stockley Park); Zimmerhackl, L.B. (Innsbruck); Rose, K. (Basel)
Points to Consider when Planning the Collection of Blood or Tissue Samples in Clinical Trials of Investigational Medicinal Products in Children, Infants and Neonates
Hawcutt, D.B.; Rose, A.C. (Liverpool); Fuerst-Recktenwald, S. (Basel); Nunn, T.; Turner, M.A. (Liverpool)
General and Specific Scientific Challenges
______________________
What Constitutes Adequate Strength of Evidence?
Day, S. (Welwyn Garden City)
Paediatric Formulations
Tuleu, C. (London); Solomonidou, D. (Basel); Breitkreutz, J. (Düsseldorf)
The Future of Oral Paediatric Formulations
Bar-Shalom, D. (Copenhagen)
Paediatric Pharmacovigilance in Clinical Research and Drug Development
Mentzer, D. (Langen); Day, S. (Welwyn Garden City)
Role of Non-Clinical Safety Assessment in Paediatric Drug Development
De Schaepdrijver, L.M. (Beerse)
Paediatric Critical Care
Goldstein, B. (Clinton, N.J./New Brunswick, N.J.); Nelson, R.M. (Silver Spring, Md.); Nadel, S. (London)
Disease-Specific Challenges
______________________
Research and Drug Development in Paediatric Oncology
Massimini, G.; Rose, K. (Basel); Vassal, G. (Villejuif); Pritchard-Jones, K. (Sutton)
Paediatric Cancer Treatment in Africa
Renner, L.A. (Accra)
Paediatric Clinical Research in Immunology and Inflammation
Bolte, C. (Basel); Senolt, L. (Prague); Gay, S. (Zurich)
Clinical Trials of Anti-Infective Agents in Paediatrics
Lutsar, I. (Tartu); Friedland, I. (Lexington, Mass.)
Obesity in Children
Hebebrand, J. (Essen)
Familial Dyslipidaemia in Children
Kusters, D.M.; Avis, H.J.; Vissers, M.N.; Hutten, B.A.; Kastelein, J.J.P.; Wiegman, A. (Amsterdam)
Paediatric Anti-Hypertensive Clinical Trials and Various Factors Influencing Trial Success or Failure
Li, J.S.; Benjamin, D.K., Jr. (Durham, N.C.); Severin, T. (Basel); Portman, R.J. (Princeton, N.J.)
Paediatric Vaccination Trials
Ceddia, F. (Wavre); Podda, A. (Siena); Vesikari, T. (Tampere)
Future of the Paediatric Pharmaceutical Market
______________________
Future of the Paediatric Pharmaceutical Market
Grek, V. (Paris); Rose, K. (Munich)
Author Index
Subject Index
Introduction
Children in the developed world have never enjoyed better medical care. Life expectancy is increasing, and child mortality is improving in most countries. However, the more we can do, the more we also see what could be done better. Every week five new genetically based rare diseases are described – for most of them there is no effective treatment and children with these diseases die early. Cancer in children is no longer untreatable. With the use of chemotherapy, radiation and surgery around 75% of the children survive. However, there are still several cancers in children where treatment is unsatisfactory, and the quality of life of many surviving children is seriously affected. The survival of children with cancer is one of the greatest success stories of medical research in the 20th century. It was not achieved by revolutionary break-through medication, but by combining the chemotherapeutic agents that reached the market in the 1940s and 1950s in new ways and new doses. From 1970 on, with each decade of diagnosis the survival of children with, e.g., acute lymphatic leukemia increased by 10-15% and is today around 90%. But in hindsight, many lives could have been saved earlier had we known how to use the existing medication. This gap was closed by systemic testing and is probably the best argument to show the value of clinical research in and with children. There are other success stories, including the advances in vaccines, surgery, nutrition, and many more.
The rapid advancement of scientific understanding of even more details of the human body combined with the availability of a large group of new genetically engineered medications has increased the desire to make these new treatment options available to children as soon as possible. This is the aim of the EU Paediatric Regulation, in force since January 2007, that has resulted in the formation of the Paediatric Committee, the strengthening of the paediatric department in the European Medicines Agency, and the submission of hundreds of Paediatric Investigation Plans by the pharmaceutical industry.
On the other hand, there are the children in developing countries that are exposed to diseases that in the majority of cases could be easily treated