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Methodologies in B iosimilar Product D evelopment Chapman & Hall/CRC Biostatistics Series Series Editors Shein-Chung Chow Duke University School of Medicine, USA Byron Jones Novartis Pharma AG, Switzerland Jen-pei Liu National Taiwan University, Taiwan Karl E. Peace Georgia Southern University, USA Bruce W. Turnbull Cornell University, USA RECENTLY PUBLISHED TITLES Single-Arm Phase II Survival Trial Design Jianrong Wu Methodologies in Biosimilar Product Development Sang Joon Lee and Shein-Chung Chow (eds.) Statistical Design and Analysis of Clinical Trials Principles and Methods, Second Edition Weichung Joe Shih and Joseph Aisner Confidence Intervals for Discrete Data in Clinical Research Vivek Pradhan, Ashis Gangopadhyay, Sandeep Menon, Cynthia Basu, and Tathagata Banerjee Statistical Thinking in Clinical Trials Michael A. Proschan Simultaneous Global New Drug Development Multi-Regional Clinical Trials after ICH E17 Edited by Gang Li, Bruce Binkowitz, William Wang, Hui Quan, and Josh Chen For more information about this series, please visit: https://www.routledge. com/Chapman—Hall-CRC-Biostatistics-Series/book-series/CHBIOSTATIS Methodologies in B iosimilar Product D evelopment Edited by Sang Joon Lee Shein-Chung Chow First edition published 2022 by CRC Press 6000 Broken Sound Parkway NW, Suite 300, Boca Raton, FL 33487-2742 and by CRC Press 2 Park Square, Milton Park, Abingdon, Oxon, OX14 4RN © 2022 Taylor & Francis Group, LLC CRC Press is an imprint of Taylor & Francis Group, LLC Reasonable eforts have been made to publish reliable data and information, but the author and publisher cannot assume responsibility for the validity of all materials or the consequences of their use. Te authors and publishers have attempted to trace the copyright holders of all material reproduced in this publication and apologize to copyright holders if permission to publish in this form has not been obtained. If any copyrighted material has not been acknowledged, please write and let us know so we may rectify it in any future reprint. Except as permitted under U.S. Copyright Law, no part of this book may be reprinted, reproduced, transmitted, or utilized in any form by any electronic, mechanical, or other means, now known or hereafter invented, including photocopying, microflming, and recording, or in any information storage or retrieval system, without written permission from the publishers. For permission to photocopy or use material electronically from this work, access www.copyright.com or contact the Copyright Clearance Center, Inc. (CCC), 222 Rosewood Drive, Danvers, MA 01923, 978-750-8400. For works that are not available on CCC, please contact mpkbookspermissions@ tandf.co.uk. Trademark notice: Product or corporate names may be trademarks or registered trademarks and are used only for identifcation and explanation without intent to infringe. Library of Congress Cataloging-in-Publication Data Names: Lee, Sang Joon, editor. | Chow, Shein-Chung, 1955- editor. Title: Methodologies in biosimilar product development / edited by Sang Joon Lee, Shein-Chung Chow, Celltrion, Inc., Incheon, Republic of Korea, Duke University School of Medicine, Durham, North Carolina, USA. Description: First edition. | Boca Raton : CRC Press, 2022. | Series: Chapman & Hall/CRC biostatistics series | Includes bibliographical references and index. Identifers: LCCN 2021021525 (print) | LCCN 2021021526 (ebook) | ISBN 9780367896348 (hardback) | ISBN 9781032071800 (paperback) | ISBN 9781003205807 (ebook) Subjects: LCSH: Drug development. | Biologicals. Classifcation: LCC RM301.25 .M46 2022 (print) | LCC RM301.25 (ebook) | DDC 615.1/9—dc23 LC record available at https://lccn.loc.gov/2021021525 LC ebook record available at https://lccn.loc.gov/2021021526 ISBN: 9780367896348 (hbk) ISBN: 9781032071800 (pbk) ISBN: 9781003205807 (ebk) DOI: 10.1201/9781003205807 Typeset in Palatino by codeMantra Contents Preface.................................................................................................................... vii Authors....................................................................................................................ix Contributors............................................................................................................xi 1. Biosimilar Product Development ................................................................1 Sang Joon Lee and Shein-Chung Chow 2. CMC Considerations for Quality ..............................................................23 Shein-Chung Chow, Sanghee Kim, and Wei Zhang 3. Quality by Design ........................................................................................43 Min Joo Choo, Ji Youn Kim, Shein-Chung Chow, and Sang Joon Lee 4. Stability Studies............................................................................................77 Mo Liu, Yuqing Liu, and Shein-Chung Chow 5. Two One-Sided Tests Versus Confdence Interval Approach .............97 Jingyu Julia Luan, Weijia Mai, and Shein-Chung Chow 6. Equivalence Test with Flexible Margin ................................................. 115 Sang Joon Lee, Minkyung Oh, and Shein-Chung Chow 7. Modifed Quality Range Method for Analytical Similarity  Evaluation................................................................................. 135 Sung Taek Son, Min Kyung Oh, Min Joo Choo, Shein-Chung Chow, and  Sang Joon Lee 8. PK/PD Bridging Studies ........................................................................... 159 Fuyu Song, Xin Zheng, and Shein-Chung Chow 9. Non-inferiority/Similarity Margin......................................................... 173 Jingyu Julia Luan, Wenyue Zhuo, and Shein-Chung Chow 10. Design and Analysis of Biosimilar Switching Studies ......................205 Shein-Chung Chow, and Sang Joon Lee 11. Detecting Reference Product Change in Biosimilar Studies ............227 Jiayin Zheng, Peijin Wang, Yixin Wang, and Shein-Chung Chow v vi Contents 12. Sensitivity Analysis for Assessment of Extrapolation .......................253 Junheng Gao, Chenxuan Zang, Ying Lyu, and Shein-Chung Chow 13. Non-Medical Switch ..................................................................................277 Fuyu Song, He Bai, Peijin Wang, and Shein-Chung Chow 14. Case Studies..................................................................................................291 Ilsung Chang, Ti Na Pyo, Seul Gi Lee, Sin Hye Kim, Min Joo Choo, and  Sang Joon Lee Index ......................................................................................................................371 Preface Biologic drug products are therapeutic moieties that are manufactured using a living system or an organism, which are important life-saving drug p roducts for patients with unmet medical needs. Biosimilars are similars that are made after the patent expiration of innovative biological products. To assist sponsors in developing biosimilar products, regulatory agencies such as the United States (US) Food and Drug Administration (FDA) have published several guidance since the BPCI Act was passed by the US Congress in 2009. Following the regu- latory guidance, several methodologies for assessment of analytical similarity, pharmacokinetic and pharmacodynamic (PK/PD) similarity, and clinical simi- larity have been developed in the literature. However, due to the complexity of the structures of biosimilar products and the nature of the manufacturing process, the development of biosimilar products is very different from that of traditional small molecule (chemical) drug products. Thus, some practical and challenging issues such as the selection of similarity margin, drug interchange- ability, non-medical switch, possible reference product change, extrapolation across different indications, etc. inevitably occur during the development of bio- similar products. Methodology development for addressing these practical and challenging issues is necessary for assuring an unbiased and reliable assessment of the treatment effect of the proposed biosimilar product under investigation. This book is intended to deal with some practical and challenging issues that are commonly encountered during the development, review, and approval of a proposed biosimilar product. These practical and challenging issues include, but are not limited to, (1) the mix-up use of interval hypoth- eses testing (i.e., the use of TOST) and confdence interval approach, (2) the detection of possible reference product change over time, (3) design and anal- ysis of biosimilar switching studies, (4) the assessment of sensitivity index for assessment of extrapolation across indications without collecting data from those indications not under study, and (5) the feasibility and valida- tion of non-medical switch post-approval. We hope that this book can serve as a bridge among the pharmaceutical/biotechnology industry, government regulatory agencies, and academia. The scope of this book is not only to cover scientifc factors and practical issues that are commonly encountered in biosimilar studies, but also to focus on recent development in research and development. This book contains 14 chapters. Chapter 1 provides an overview of biosimilar product research and development. Also included in this chapter is a brief instruction of some com- monly seen scientifc factors/practical issues and recent methodology devel- opment in biosimilar product research and development. Chapter 2 reviews chemistry, manufacturing, and control (CMC) requirements including man- ufacturing process validation, quality control and assurance, and stability vii viii Preface analysis for a biosimilar product. Chapter 3 introduces the concept and the use of quality by design in biosimilar product development. Statistical design and analysis of stability studies for drug shelf-life estimation are discussed in Chapter 4. Chapter 5 discusses the relationship between the method of inter- val hypotheses testing and the confdence interval approach for similarity assessment in biosimilar studies. An effcient equivalence test with a fexible margin for analytical similarity assessment is proposed in Chapter 6 followed by an evaluation of the FDA recommended method of quality range (QR) for analytical similarity assessment in Chapter 7. Also included in Chapter 7 is a proposed method of modifed QR (mQR) for analytical similarity assessment. Chapter 8 reviews various statistical designs for pharmacokinetic and phar- macodynamic (PK/PD) bridging studies with multiple reference products from different regions such as European Union and the United States. Chapter 9 proposes a method based on the evaluation of relative risk/beneft for selec- tion of similarity margin for similarity assessment when there is a disagree- ment between the sponsors and regulatory agencies. Chapter 10 provides an overview of switching studies for addressing drug interchangeability in terms of the assessment of risk with/without switching and alternation. The issue of a potential reference product change in b iosimilar studies is discussed in Chapter 11. Chapter 12 studies the issue of extrapolation across indications (the conditions of use) and proposes statistical methods for assessment of extrapo- lation in terms of sensitivity index for generalizability between patient popula- tions (related to conditions of use). Chapter 13 provides statistical considerations for the assessment of no-medical switch in biosimilar studies. Chapter 14 dis- cusses some case studies concerning recent regulatory submissions. We are indebted to many people for the completion of this book. From Taylor & Francis, we offer our thanks to Mr. David Grubbs for providing us with the opportunity to work on this book project. We also thank colleagues from Celltrion, Inc. and Duke University School of Medicine and many friends from the academia, the pharmaceutical industry, and, regulatory agen- cies including US FDA, EU EMA, CDE/NMPA of China, and MFDS of the Republic of Korea for their support, encouragement, and discussions during the preparation of this book. The frst editor would like to express his appre- ciation to Mr. Woo-Sung Kee, the CEO of Celltrion for his encouragement and friendship since his frst day at Celltrion, and Dr. Jung-Jin Seo, the founder and the honorary chairman of Celltrion, for his perspicacity and leadership of bio- similar product development. Lastly, the frst editor also would like to thank his loving wife, Sun-Ah, and his lovely daughters Alicia Kayoung and Sophia Jooyoung for their support during the preparation of this book manuscript. Finally, the views expressed are those of the editors and not necessarily those of Celltrion and Duke University School of Medicine. We are solely responsible for the contents and errors of this book. Any comments and suggestions for future editions of this book are very much appreciated. Sang Joon Lee, PhD Shein-Chung Chow, PhD Authors Sang Joon Lee, Ph.D., is a Senior Executive Vice President at Celltrion. Dr. Lee was previously a biostatistician at academia including the University of New Mexico School of Medicine and The University of Texas, M.D. Anderson Cancer Center. He received a Ph.D. degree in Statistics from Texas A&M University. Dr. Lee has authored or co-authored over 100 research papers and invited papers. Shein-Chung Chow, Ph.D., is a Professor at the Duke University School of Medicine. Dr. Chow was previously the Associate Director at the Offce of Biostatistics, Center for Drug Evaluation and Research, United States Food and Drug Administration (FDA). Dr. Chow was also at Millennium, Covance, and Bristol-Myers Squibb. He received a Ph.D. degree in Statistics from the University of Wisconsin-Madison. Dr. Chow has authored or co-authored over 310 methodology papers and 31 books. ix

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