INTERNATIONAL COOPERATION, CONVERGENCE, AND HARMONIZATION OF PHARMACEUTICAL REGULATIONS A GLOBAL PERSPECTIVE PIERRE-LOUIS LEZOTRE, MS, PhD To contact the Author: [email protected] AMSTERDAM • BOSTON • HEIDELBERG • LONDON NEW YORK • OXFORD • PARIS • SAN DIEGO SAN FRANCISCO • SINGAPORE • SYDNEY • TOKYO Academic Press is an imprint of Elsevier Academic Press is an imprint of Elsevier The Boulevard, Langford Lane, Kidlington, Oxford, OX5 1GB 525 B Street, Suite 1800, San Diego, CA 92101-4495, USA First published 2014 Copyright © 2014 Pierre-Louis Lezotre. Published by Elsevier Inc. All rights reserved. The right of Pierre-Louis Lezotre to be identified as the author of this work has been asserted in accordance with the Copyright, Designs and Patents Act 1988. No part of this publication may be reproduced or transmitted in any form or by any means, electronic or mechanical, including photocopying, recording, or any information storage and retrieval system, without permission in writing from the publisher. Details on how to seek permission, further information about the Publisher’s permissions policies and our arrangement with organizations such as the Copyright Clearance Center and the Copyright Licensing Agency, can be found at our website: www.elsevier.com/permissions Notices Knowledge and best practice in this field are constantly changing. As new research and experience broaden our understanding, changes in research methods, professional practices, or medical treatment may become necessary. Practitioners and researchers must always rely on their own experience and knowledge in evaluating and using any information, methods, compounds, or experiments described herein. In using such information or methods they should be mindful of their own safety and the safety of others, including parties for whom they have a professional responsibility. To the fullest extent of the law, neither the Publisher nor the authors, contributors, or editors, assume any liability for any injury and/or damage to persons or property as a matter of products liability, negligence or otherwise, or from any use or operation of any methods, products, instructions, or ideas contained in the material herein. British Library Cataloguing in Publication Data A catalogue record for this book is available from the British Library Library of Congress Cataloguing in Publication Data A catalog record for this book is available from the Library of Congress ISBN: 978-0-12-800053-3 For information on all Academic Press publications visit our website at store.elsevier.com Printed and bound in the United States 14 15 16 17 10 9 8 7 6 5 4 3 2 1 To Isabelle, Cléo and Noah “Wisdom is to have dreams that are big enough not to lose sight when we pursue them.” Oscar Wilde Preface The preparation of this book has been a long journey! It is the outcome of 5 years of serious research and reflection on cooperation, convergence and harmonization in the pharmaceutical domain. In fact, it is even more than that. It represents the knowledge acquired during 15 years of practical experience as a professional involved in global phar- maceutical regulation and, even more importantly, the result of many discussions with experts and friends on this fascinating topic. Many people contributed to this book in numerous ways, and I am grateful to all of them. My several professional positions, in both Europe and the United States, allowed me to meet many people that shared my passion for this topic. I particularly remember certain “debates” with Ms. Marie Dray in Washington DC or a late evening phone conversation with Dr. Murray Lumpkin where we exchanged points of view on this subject. These discussions stimulated me to further explore this particular topic and to finally write a book on it! I would like to sincerely thank and acknowledge each of the following people who helped me in the preparation of this document: - Mr. Maher Aljaser, MSc, Senior Pharmacist, Licensing Department, Drug Sector, Saudi Food and Drug Authority, Riyadh, Saudi Arabia - Dr. Samvel Azatyan MD, PhD, Manager, Medicines Regulatory Support Programme [MRS], Quality Assurance and Safety: Medicines, Department of Essential Medicines and Health Products (EMP), World Health Organization, Switzerland - Prof. Saleh A. Bawazir, PhD, Vice President for Drug Affairs, Saudi Food and Drug Authority, Riyadh, Saudi Arabia - Mrs. Hilde Boone, European Medicines Agency Liaison Official at the US FDA, Silver Spring, Maryland, United States of America - Prof. Sir Alasdair Breckenridge, CBE, MD, Chairman of the Board of the MHRA, London, United Kingdom - Ms. Marie Allison Dray, MBA, President of International Regulatory Affairs Group, LLC, Naples, Florida, United States of America - Dr. Hajed M. Hashan, PhD, Executive Director of Licensing Department, Drug Sector, Saudi Food and Drug Authority, Riyadh, Saudi Arabia - Dr. Christina Lim, PhD, Senior Director, International Relations, Health Products Regulation Group, Health Sciences Authority, Singapore - Dr. Murray M. Lumpkin, MD, Commissioner’s Senior Advisor and Representative for Global Issues, US FDA, Silver Spring, Maryland, United States of America xi xii PREFACE - CAPT Justina A. Molzon, MS Pharm., JD, Associate Center Director for International Programs, Center for Drug Evaluation and Research, US FDA, Silver Spring, Maryland, United States of America - Mr. Joseph Mthetwa, MSc (Medicine), Senior Programme Officer for Health and Pharmaceuticals, Directorate of Social, Human Development and Special Programmes, SADC, Gaborone, Botswana - Ms. Margareth Ndomondo-Sigonda, MBA, MSc, Pharmaceutical Coordinator, African Union-NEPAD Planning and Coordinating Agency, Johannesburg, South Africa - Prof. Jean-Yves Pabst, PhD, PharmD, Professeur de droit et économie pharmaceutique, Doyen de la Faculté de Pharmacie de Strasbourg, Strasbourg, France - Prof. Anne-Catherine Perroy Maillols, PhD, PharmD, Professeur de droit et économie pharmaceutique, Faculté de Pharmacie, Université Lille 2, Lille, France - Dr. Lembit Rago, MD, PhD, Coordinator, Quality Assurance and Safety: Medicines, Essential Medicines and Pharmaceutical Policies, WHO, Geneva, Switzerland - Dr. Tomas Salmonson, PhD, CHMP Chair, European Medicines Agency, London, United Kingdom - Dr. Dean K. Smith, PhD, Senior Evaluator, Bacterial & Combination Vaccines Division, Biologics and Genetic Therapies Directorate, Health Canada, Ottawa, Canada - Mrs. Marie Tham, Advisor, International Collaboration, Health Products Regulation Group, Health Sciences Authority, Singapore - Prof. Daniel Vion, PharmD, Doyen de la Faculté de Pharmacie de Lille, Lille, France - Mr. Mike Ward, Manager International Programs at Health Canada and APEC RHSC Chair, Ottawa, Canada - Prof. Stuart Walker, PhD, Professor of Pharmaceutical Medicine, School of Pharmacy, Cardiff University, Wales & Founder of CMR International & the Centre for Innovation in Regulatory Science, London, United Kingdom - Dr. Cheryl Watton, MD, MBA, VP, Head of Global Safety & Epidemiology, Allergan Inc., Irvine, California, United States of America This list includes the best international experts in the field, representing academia, regulatory authorities, and industry. Most of them have been dedicated to international cooperation and harmo- nization of pharmaceutical regulations for many years. They are well recognized for their incredible efforts and significant value added to several bilateral, regional, and/or global harmonization initia- tives. However, despite their significant responsibilities and busy schedules, they did not hesitate and accepted with enthusiasm the opportunity to assist me. The support from all of these international experts allowed me to have accurate and up- to-date nonproduct-related information on the ongoing worldwide harmonization and cooperation activities (Part I of this document). Follow-up discussions with some of them have also been extremely beneficial. It helped me to further develop my analysis on the matter, to challenge my ideas and proposal so that I could improve them, and to focus on the critical items. Their expert support, enthusiasm, and kindness were very much appreciated. PREFACE xiii I would also like to express my sincere gratitude to Dr. Susan Garabedian-Ruffalo and Ms. Barbara Swenson, my two linguistic experts! The content and accuracy of a scientific or technical book is obviously important, but the form, the way you express and structure your ideas, and the clarity of the explanation and argumentation are critical. Susan and Barbara’s unconditional continuous support and constructive advice over the past years have added significant value to this book! I hope my work will bring another piece to the puzzle and contribute to the increase of cooperation in the pharmaceutical domain. I am looking forward to further exchanges and debates on this fascinating and important subject, which is critical to protect and promote global public health! Pierre-Louis Lezotre, MS, PhD. About the Author Doctor Pierre-Louis Lezotre, MS, PhD, is presently Director of Global Regulatory Affairs at Allergan Inc., California, USA. He specializes in global regulatory strategy, and is recognized for his passion and expertise on international cooperation, con- vergence, and harmonization of regulations for pharmaceutical and biotechnology products. He has worked in different cultural environments and lived in both Europe and the United States. Dr. Lezotre studied biology (University of Sciences, Saint- Etienne, France) and drug development (University