Clinical Pharmacology: Current Topics and Case Studies

Clinical Pharmacology: Current Topics and Case Studies

Markus Müller Editor Clinical Pharmacology: Current Topics and Case Studies Second Edition

Editor Markus Müller Universitätsklinik für Klinische Pharmakologie Medizinische Universität Wien Wien Austria ISBN 978-3-319-27345-7 DOI 10.1007/978-3-319-27347-1 ISBN 978-3-319-27347-1 (ebook) Library of Congress Control Number: 2016935068 Springer Cham Heidelberg New York Dordrecht London Springer International Publishing Switzerland 2016 This work is subject to copyright. All rights are reserved by the Publisher, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission or information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed. The use of general descriptive names, registered names, trademarks, service marks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. The publisher, the authors and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authors or the editors give a warranty, express or implied, with respect to the material contained herein or for any errors or omissions that may have been made. Printed on acid-free paper Springer International Publishing AG Switzerland is part of Springer Science+Business Media (www. springer.com)

Contents Part I Introduction 1 The Discipline of Clinical Pharmacology......................... 3 Markus Müller 2 Current Issues in Drug Development............................ 7 Markus Müller 3 Current Issues in Drug Regulation............................. 19 Christa Wirthumer-Hoche and Brigitte Bloechl-Daum 4 Current Topics in Drug Reimbursement........................ 33 Anna Bucsics, Robert Sauermann, and Valerie Nell-Duxneuner Part II Clinical Trials 5 Ethics in Clinical Research.................................... 51 Ernst Singer and Christiane Druml 6 Good Clinical Practice (GCP) and Scientific Misconduct........... 61 Brigitte Bloechl-Daum 7 Phase I Studies and First-In-Human Trials...................... 85 Ulla Derhaschnig and Bernd Jilma 8 Clinical Trials: Interventional Studies.......................... 97 Michael Wolzt and Stefan Aschauer 9 Observational Studies....................................... 109 Harald Herkner and Christoph Male Part III Tools in Clinical Pharmacology 10 Tools in Clinical Pharmacology: Imaging Techniques............ 139 Martin Bauer and Oliver Langer 11 Pharmacokinetics II: 14 C-Labelled Microdosing in Assessing Drug Pharmacokinetics at Phase 0................. 151 Graham Lappin v

vi Contents 12 Current Concepts of Pharmacogenetics, Pharmacogenomics, and the Druggable Genome................................ 161 Wolfgang M. Schmidt and Robert M. Mader 13 Pharmacokinetics I: PK-PD Approach, the Case of Antibiotic Drug Development.............................. 185 Sherwin K.B. Sy and Hartmut Derendorf 14 Epidemiology and Biostatistics............................... 219 Gerhard Garhöfer and Leopold Schmetterer 15 Placebo Effects and Placebo Control in Clinical Trials............ 233 Johannes Pleiner-Duxneuner Part IV Topics in Clinical Pharmacology 16 Pharmaceutical Drug Safety................................. 247 Martin Brunner 17 Drug Interactions.......................................... 265 Markus Zeitlinger 18 Development of Advanced Therapy Medicinal Products: A Case for Early Scientific Advice............................ 293 Martin Brunner and Bernd Jilma 19 Biologics.................................................. 305 Bernd Jilma and Markus Müller 20 Generics, Biosimilars, Enantiomers, and Me-Toos............... 317 Dieter Deforce, George Wade, and Brigitte Bloechl-Daum 21 Orphan Drugs for Rare Diseases.............................. 337 Brigitte Bloechl-Daum, Florence Butlen-Ducuing, and Jordi Llinares-Garcia 22 Special Situations, Market Fragmentation II: Sex Differences..... 361 Ghazaleh Gouya 23 Special Situations III: Medicines for Children................... 377 Christoph Male About the Authors.............................................. 393 Index......................................................... 401