Statistical methods for immunogenicity assessment PDF
Preview Statistical methods for immunogenicity assessment
Statistics S t a t i s Immunogenicity assessment is a prerequisite for the successful de- t i c velopment of biopharmaceuticals, including safety and efficacy eval- a l uation. Using advanced statistical methods in the study design and M Statistical analysis stages is therefore essential to immunogenicity risk assess- e t ment and mitigation strategies. Statistical Methods for Immunoge- h Methods for o nicity Assessment provides a single source of information on sta- d s tistical concepts, principles, methods, and strategies for detection, f Immunogenicity o quantification, assessment, and control of immunogenicity. r I The book first gives an overview of the impact of immunogenicity on m Assessment m biopharmaceutical development, regulatory requirements, and sta- u tistical methods and strategies used for immunogenicity detection, n o quantification, and risk assessment and mitigation. It then covers g e anti-drug antibody (ADA) assay development, optimization, valida- n tion, and transfer as well as the analysis of cut point, a key assay ic i performance parameter in ADA assay development and validation. t y The authors illustrate how to apply statistical modeling approach- A s es to establish associations between ADA and clinical outcomes, s e predict immunogenicity risk, and develop risk mitigation strategies. s s They also present various strategies for immunogenicity risk control. m The book concludes with an explanation of the computer codes and e n algorithms of the statistical methods. t A critical issue in the development of biologics, immunogenicity can Y cause early termination or limited use of the products if not managed a n well. This book shows how to use robust statistical methods for de- g • tecting, quantifying, assessing, and mitigating immunogenicity risk. Z It is an invaluable resource for anyone involved in immunogenicity h a n risk assessment and control in both non-clinical and clinical biophar- g • maceutical development. Y u Harry Yang • Jianchun Zhang • Z h Binbing Yu • Wei Zhao a o K24516 ISBN: 978-1-4987-0034-4 90000 9 781498 700344 www.crcpress.com K24516_cover.indd 1 6/3/15 10:15 AM Statistical Methods for Immunogenicity Assessment Editor-in-Chief Shein-Chung Chow, Ph.D., Professor, Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina Series Editors Byron Jones, Biometrical Fellow, Statistical Methodology, Integrated Information Sciences, Novartis Pharma AG, Basel, Switzerland Jen-pei Liu, Professor, Division of Biometry, Department of Agronomy, National Taiwan University, Taipei, Taiwan Karl E. Peace, Georgia Cancer Coalition, Distinguished Cancer Scholar, Senior Research Scientist and Professor of Biostatistics, Jiann-Ping Hsu College of Public Health, Georgia Southern University, Statesboro, Georgia Bruce W. Turnbull, Professor, School of Operations Research and Industrial Engineering, Cornell University, Ithaca, New York Published Titles Adaptive Design Methods in Bayesian Methods in Epidemiology Clinical Trials, Second Edition Lyle D. Broemeling Shein-Chung Chow and Mark Chang Bayesian Methods in Health Economics Adaptive Designs for Sequential Gianluca Baio Treatment Allocation Bayesian Missing Data Problems: EM, Alessandro Baldi Antognini and Data Augmentation and Noniterative Alessandra Giovagnoli Computation Adaptive Design Theory and Ming T. Tan, Guo-Liang Tian, Implementation Using SAS and R, and Kai Wang Ng Second Edition Bayesian Modeling in Bioinformatics Mark Chang Dipak K. Dey, Samiran Ghosh, Advanced Bayesian Methods for Medical and Bani K. Mallick Test Accuracy Benefit-Risk Assessment in Lyle D. Broemeling Pharmaceutical Research and Advances in Clinical Trial Biostatistics Development Nancy L. Geller Andreas Sashegyi, James Felli, and Rebecca Noel Applied Meta-Analysis with R Ding-Geng (Din) Chen and Karl E. Peace Biosimilars: Design and Analysis of Follow-on Biologics Basic Statistics and Pharmaceutical Shein-Chung Chow Statistical Applications, Second Edition James E. De Muth Biostatistics: A Computing Approach Stewart J. Anderson Bayesian Adaptive Methods for Clinical Trials Causal Analysis in Biomedicine and Scott M. Berry, Bradley P. Carlin, Epidemiology: Based on Minimal J. Jack Lee, and Peter Muller Sufficient Causation Mikel Aickin Bayesian Analysis Made Simple: An Excel GUI for WinBUGS Clinical and Statistical Considerations Phil Woodward in Personalized Medicine Claudio Carini, Sandeep Menon, Bayesian Methods for Measures of and Mark Chang Agreement Lyle D. Broemeling Clinical Trial Data Analysis using R DNA Methylation Microarrays: Ding-Geng (Din) Chen and Karl E. Peace Experimental Design and Statistical Analysis Clinical Trial Methodology Sun-Chong Wang and Arturas Petronis Karl E. Peace and Ding-Geng (Din) Chen DNA Microarrays and Related Genomics Computational Methods in Biomedical Techniques: Design, Analysis, and Research Interpretation of Experiments Ravindra Khattree and Dayanand N. Naik David B. Allison, Grier P. Page, Computational Pharmacokinetics T. Mark Beasley, and Jode W. Edwards Anders Källén Dose Finding by the Continual Confidence Intervals for Proportions and Reassessment Method Related Measures of Effect Size Ying Kuen Cheung Robert G. Newcombe Elementary Bayesian Biostatistics Controversial Statistical Issues in Lemuel A. Moyé Clinical Trials Empirical Likelihood Method in Survival Shein-Chung Chow Analysis Data Analysis with Competing Risks and Mai Zhou Intermediate States Exposure-Response Modeling: Methods Ronald B. Geskus and Practical Implementation Data and Safety Monitoring Committees Jixian Wang in Clinical Trials Frailty Models in Survival Analysis Jay Herson Andreas Wienke Design and Analysis of Animal Studies in Generalized Linear Models: A Bayesian Pharmaceutical Development Perspective Shein-Chung Chow and Jen-pei Liu Dipak K. Dey, Sujit K. Ghosh, Design and Analysis of Bioavailability and and Bani K. Mallick Bioequivalence Studies, Third Edition Handbook of Regression and Modeling: Shein-Chung Chow and Jen-pei Liu Applications for the Clinical and Design and Analysis of Bridging Studies Pharmaceutical Industries Jen-pei Liu, Shein-Chung Chow, Daryl S. Paulson and Chin-Fu Hsiao Inference Principles for Biostatisticians Design and Analysis of Clinical Trials for Ian C. Marschner Predictive Medicine Interval-Censored Time-to-Event Data: Shigeyuki Matsui, Marc Buyse, Methods and Applications and Richard Simon Ding-Geng (Din) Chen, Jianguo Sun, Design and Analysis of Clinical Trials with and Karl E. Peace Time-to-Event Endpoints Introductory Adaptive Trial Designs: Karl E. Peace A Practical Guide with R Design and Analysis of Non-Inferiority Mark Chang Trials Joint Models for Longitudinal and Time- Mark D. Rothmann, Brian L. Wiens, to-Event Data: With Applications in R and Ivan S. F. Chan Dimitris Rizopoulos Difference Equations with Public Health Measures of Interobserver Agreement Applications and Reliability, Second Edition Lemuel A. Moyé and Asha Seth Kapadia Mohamed M. Shoukri Medical Biostatistics, Third Edition Sample Size Calculations for Clustered A. Indrayan and Longitudinal Outcomes in Clinical Research Meta-Analysis in Medicine and Health Chul Ahn, Moonseong Heo, and Policy Song Zhang Dalene Stangl and Donald A. Berry Sample Size Calculations in Clinical Mixed Effects Models for the Population Research, Second Edition Approach: Models, Tasks, Methods and Shein-Chung Chow, Jun Shao Tools and Hansheng Wang Marc Lavielle Statistical Analysis of Human Growth Modeling to Inform Infectious Disease and Development Control Yin Bun Cheung Niels G. Becker Statistical Design and Analysis of Modern Adaptive Randomized Clinical Stability Studies Trials: Statistical and Practical Aspects Shein-Chung Chow Oleksandr Sverdlov Statistical Evaluation of Diagnostic Monte Carlo Simulation for the Performance: Topics in ROC Analysis Pharmaceutical Industry: Concepts, Kelly H. Zou, Aiyi Liu, Andriy Bandos, Algorithms, and Case Studies Lucila Ohno-Machado, and Howard Rockette Mark Chang Statistical Methods for Clinical Trials Multiple Testing Problems in Mark X. Norleans Pharmaceutical Statistics Alex Dmitrienko, Ajit C. Tamhane, Statistical Methods for Drug Safety and Frank Bretz Robert D. Gibbons and Anup K. Amatya Noninferiority Testing in Clinical Trials: Statistical Methods for Immunogenicity Issues and Challenges Assessment Tie-Hua Ng Harry Yang, Jianchun Zhang, Binbing Yu, and Wei Zhao Optimal Design for Nonlinear Response Models Statistical Methods in Drug Combination Valerii V. Fedorov and Sergei L. Leonov Studies Wei Zhao and Harry Yang Patient-Reported Outcomes: Measurement, Implementation and Statistics in Drug Research: Interpretation Methodologies and Recent Joseph C. Cappelleri, Kelly H. Zou, Developments Andrew G. Bushmakin, Jose Ma. J. Alvir, Shein-Chung Chow and Jun Shao Demissie Alemayehu, and Tara Symonds Statistics in the Pharmaceutical Industry, Quantitative Evaluation of Safety in Drug Third Edition Development: Design, Analysis and Ralph Buncher and Jia-Yeong Tsay Reporting Survival Analysis in Medicine and Qi Jiang and H. Amy Xia Genetics Randomized Clinical Trials of Jialiang Li and Shuangge Ma Nonpharmacological Treatments Theory of Drug Development Isabelle Boutron, Philippe Ravaud, and Eric B. Holmgren David Moher Translational Medicine: Strategies and Randomized Phase II Cancer Clinical Statistical Methods Trials Dennis Cosmatos and Shein-Chung Chow Sin-Ho Jung Statistical Methods for Immunogenicity Assessment Harry Yang • Jianchun Zhang Binbing Yu • Wei Zhao MedImmune, LLC Gaithersburg, Maryland, USA CRC Press Taylor & Francis Group 6000 Broken Sound Parkway NW, Suite 300 Boca Raton, FL 33487-2742 © 2016 by Taylor & Francis Group, LLC CRC Press is an imprint of Taylor & Francis Group, an Informa business No claim to original U.S. Government works Version Date: 20150515 International Standard Book Number-13: 978-1-4987-0035-1 (eBook - PDF) This book contains information obtained from authentic and highly regarded sources. Reasonable efforts 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. The 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 copyright material has not been acknowledged please write and let us know so we may rectify 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, microfilming, and recording, or in any information stor- age or retrieval system, without written permission from the publishers. For permission to photocopy or use material electronically from this work, please access www.copy- right.com (http://www.copyright.com/) or contact the Copyright Clearance Center, Inc. (CCC), 222 Rosewood Drive, Danvers, MA 01923, 978-750-8400. CCC is a not-for-profit organization that pro- vides licenses and registration for a variety of users. For organizations that have been granted a photo- copy license by the CCC, a separate system of payment has been arranged. Trademark Notice: Product or corporate names may be trademarks or registered trademarks, and are used only for identification and explanation without intent to infringe. Visit the Taylor & Francis Web site at http://www.taylorandfrancis.com and the CRC Press Web site at http://www.crcpress.com To our families TThhiiss ppaaggee iinntteennttiioonnaallllyy lleefftt bbllaannkk Contents Preface xv List of Figures xvii List of Tables xxi 1 Introduction 1 1.1 Background . . . . . . . . . . . . . . . . . . . . . . . . . . . 2 1.2 Immunogenicity . . . . . . . . . . . . . . . . . . . . . . . . . 4 1.3 Impact of Immunogenicity . . . . . . . . . . . . . . . . . . . 5 1.4 Regulatory Environment and Guidelines . . . . . . . . . . . 6 1.4.1 FDA Guidelines . . . . . . . . . . . . . . . . . . . . . 7 1.4.1.1 Tiered Approach to ADA Assay Development 7 1.4.1.2 Immunogenicity Risk Assessment . . . . . . 9 1.4.2 European Medicines Agency (EMA) Guidance . . . . 13 1.4.2.1 EMA Guidelines on Immunogenicity Assessment . . . . . . . . . . . . . . . . . . 13 1.4.2.2 Latest Development of EMA Immunogenicity Guidelines . . . . . . . . . . . . . . . . . . . 14 1.4.3 Japanese Regulatory Requirements of Immunogenicity 14 1.5 Statistics in Immunogenicity Risk Assessment . . . . . . . . 15 1.5.1 In Silico Prediction of Immunogenicity . . . . . . . . . 16 1.5.2 ADA Detection and Quantification . . . . . . . . . . . 16 1.5.3 Clinical Characterization of ADA . . . . . . . . . . . . 17 1.5.3.1 Characteristics of ADA Immune Response . 17 1.5.3.2 Correlation between ADA and PK/PD, Clinical Safety and Efficacy . . . . . . . . . . 18 1.5.3.3 Relationship of ADA with Clinical Efficacy and Safety . . . . . . . . . . . . . . . . . . . 18 1.5.3.4 Identification of Risk Factors . . . . . . . . . 18 1.5.4 Control of Immunogenicity Risk . . . . . . . . . . . . 19 1.5.4.1 Control of Process/Product Factors . . . . . 19 1.5.4.2 Biomarkers for Immunogenicity . . . . . . . 19 1.6 Statistical Considerations in Comparative Immunogenicity Studies . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20 ix
The list of books you might like
