Design analysis of clinical trials for economic evaluation reimbursement

Design & Analysis of Clinical Trials for Economic Evaluation & Reimbursement An Applied Approach Using SAS & STATA S         Iftekhar Khan Design & Analysis of Clinical Trials for Economic Evaluation & Reimbursement An Applied Approach Using SAS & STATA 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 Clinical Trials, Second Edition Shein-Chung Chow and Mark Chang Bayesian Analysis Made Simple: An Excel GUI for WinBUGS Phil Woodward Adaptive Designs for Sequential Treatment Allocation Alessandro Baldi Antognini and Alessandra Giovagnoli Bayesian Methods for Measures of Agreement Lyle D Broemeling Adaptive Design Theory and Implementation Using SAS and R, Second Edition Mark Chang Advanced Bayesian Methods for Medical Test Accuracy Lyle D Broemeling Advances in Clinical Trial Biostatistics Nancy L Geller Applied Meta-Analysis with R Ding-Geng (Din) Chen and Karl E Peace Basic Statistics and Pharmaceutical Statistical Applications, Second Edition James E De Muth Bayesian Adaptive Methods for Clinical Trials Scott M Berry, Bradley P Carlin, J Jack Lee, and Peter Muller Bayesian Methods for Repeated Measures Lyle D Broemeling Bayesian Methods in Epidemiology Lyle D Broemeling Bayesian Methods in Health Economics Gianluca Baio Bayesian Missing Data Problems: EM, Data Augmentation and Noniterative Computation Ming T Tan, Guo-Liang Tian, and Kai Wang Ng Bayesian Modeling in Bioinformatics Dipak K Dey, Samiran Ghosh, and Bani K Mallick Benefit-Risk Assessment in Pharmaceutical Research and Development Andreas Sashegyi, James Felli, and Rebecca Noel Published Titles Biosimilars: Design and Analysis of Follow-on Biologics Shein-Chung Chow Biostatistics: A Computing Approach Stewart J Anderson Causal Analysis in Biomedicine and Epidemiology: Based on Minimal Sufficient Causation Mikel Aickin Clinical and Statistical Considerations in Personalized Medicine Claudio Carini, Sandeep Menon, and Mark Chang Clinical Trial Data Analysis using R Ding-Geng (Din) Chen and Karl E Peace Clinical Trial Methodology Karl E Peace and Ding-Geng (Din) Chen Computational Methods in Biomedical Research Ravindra Khattree and Dayanand N Naik Computational Pharmacokinetics Anders Källén Confidence Intervals for Proportions and Related Measures of Effect Size Robert G Newcombe Controversial Statistical Issues in Clinical Trials Shein-Chung Chow Data Analysis with Competing Risks and Intermediate States Ronald B Geskus Data and Safety Monitoring Committees in Clinical Trials Jay Herson Design and Analysis of Animal Studies in Pharmaceutical Development Shein-Chung Chow and Jen-pei Liu Design and Analysis of Bioavailability and Bioequivalence Studies, Third Edition Shein-Chung Chow and Jen-pei Liu Design and Analysis of Bridging Studies Jen-pei Liu, Shein-Chung Chow, and Chin-Fu Hsiao Design & Analysis of Clinical Trials for Economic Evaluation & Reimbursement : An Applied Approach Using SAS & STATA Iftekhar Khan Design and Analysis of Clinical Trials for Predictive Medicine Shigeyuki Matsui, Marc Buyse, and Richard Simon Design and Analysis of Clinical Trials with Time-to-Event Endpoints Karl E Peace Design and Analysis of Non-Inferiority Trials Mark D Rothmann, Brian L Wiens, and Ivan S F Chan Difference Equations with Public Health Applications Lemuel A Moyé and Asha Seth Kapadia DNA Methylation Microarrays: Experimental Design and Statistical Analysis Sun-Chong Wang and Arturas Petronis Published Titles DNA Microarrays and Related Genomics Techniques: Design, Analysis, and Interpretation of Experiments David B Allison, Grier P Page, T Mark Beasley, and Jode W Edwards Dose Finding by the Continual Reassessment Method Ying Kuen Cheung Dynamical Biostatistical Models Daniel Commenges and Hélène Jacqmin-Gadda Elementary Bayesian Biostatistics Lemuel A Moyé Empirical Likelihood Method in Survival Analysis Mai Zhou Interval-Censored Time-to-Event Data: Methods and Applications Ding-Geng (Din) Chen, Jianguo Sun, and Karl E Peace Introductory Adaptive Trial Designs: A Practical Guide with R Mark Chang Joint Models for Longitudinal and Time-to-Event Data: With Applications in R Dimitris Rizopoulos Measures of Interobserver Agreement and Reliability, Second Edition Mohamed M Shoukri Medical Biostatistics, Third Edition A Indrayan Exposure–Response Modeling: Methods and Practical Implementation Jixian Wang Meta-Analysis in Medicine and Health Policy Dalene Stangl and Donald A Berry Frailty Models in Survival Analysis Andreas Wienke Mixed Effects Models for the Population Approach: Models, Tasks, Methods and Tools Marc Lavielle Fundamental Concepts for New Clinical Trialists Scott Evans and Naitee Ting Generalized Linear Models: A Bayesian Perspective Dipak K Dey, Sujit K Ghosh, and Bani K Mallick Handbook of Regression and Modeling: Applications for the Clinical and Pharmaceutical Industries Daryl S Paulson Inference Principles for Biostatisticians Ian C Marschner Modeling to Inform Infectious Disease Control Niels G Becker Modern Adaptive Randomized Clinical Trials: Statistical and Practical Aspects Oleksandr Sverdlov Monte Carlo Simulation for the Pharmaceutical Industry: Concepts, Algorithms, and Case Studies Mark Chang Multiple Testing Problems in Pharmaceutical Statistics Alex Dmitrienko, Ajit C Tamhane, and Frank Bretz Published Titles Noninferiority Testing in Clinical Trials: Issues and Challenges Tie-Hua Ng Optimal Design for Nonlinear Response Models Valerii V Fedorov and Sergei L Leonov Statistical Design and Analysis of Clinical Trials: Principles and Methods Weichung Joe Shih and Joseph Aisner Statistical Design and Analysis of Stability Studies Shein-Chung Chow Patient-Reported Outcomes: Measurement, Implementation and Interpretation Joseph C Cappelleri, Kelly H Zou, Andrew G Bushmakin, Jose Ma J Alvir, Demissie Alemayehu, and Tara Symonds Statistical Evaluation of Diagnostic Performance: Topics in ROC Analysis Kelly H Zou, Aiyi Liu, Andriy Bandos, Lucila Ohno-Machado, and Howard Rockette Quantitative Evaluation of Safety in Drug Development: Design, Analysis and Reporting Qi Jiang and H Amy Xia Statistical Methods for Drug Safety Robert D Gibbons and Anup K Amatya Statistical Methods for Clinical Trials Mark X Norleans Quantitative Methods for Traditional Chinese Medicine Development Shein-Chung Chow Statistical Methods for Immunogenicity Assessment Harry Yang, Jianchun Zhang, Binbing Yu, and Wei Zhao Randomized Clinical Trials of Nonpharmacological Treatments Isabelle Boutron, Philippe Ravaud, and David Moher Statistical Methods in Drug Combination Studies Wei Zhao and Harry Yang Randomized Phase II Cancer Clinical Trials Sin-Ho Jung Sample Size Calculations for Clustered and Longitudinal Outcomes in Clinical Research Chul Ahn, Moonseong Heo, and Song Zhang Sample Size Calculations in Clinical Research, Second Edition Shein-Chung Chow, Jun Shao, and Hansheng Wang Statistical Analysis of Human Growth and Development Yin Bun Cheung Statistics in Drug Research: Methodologies and Recent Developments Shein-Chung Chow and Jun Shao Statistics in the Pharmaceutical Industry, Third Edition Ralph Buncher and Jia-Yeong Tsay Survival Analysis in Medicine and Genetics Jialiang Li and Shuangge Ma Theory of Drug Development Eric B Holmgren Translational Medicine: Strategies and Statistical Methods Dennis Cosmatos and Shein-Chung Chow Design & Analysis of Clinical Trials for Economic Evaluation & Reimbursement An Applied Approach Using SAS & STATA Iftekhar Khan University College London, UK 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: 20150722 International Standard Book Number-13: 978-1-4665-0548-3 (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 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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 290 References Glick HA, Doshi JA, Sonnad SS, Polsky D Oxford: Oxford University Press, 2014 Goodwin PJ Reversible ovarian ablation or chemotherapy: Are we ready for quality of life to guide adjuvant treatment decisions in breast cancer? 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14(1): 93–9 Yang X, Shoptaw S, Nie K, Liu J, Belin TR Markov transition models for binary repeated measures with ignorable and nonignorable missing values Stat Methods Med Res 2007; 16(4): 347–64 Zhao H, Bang H, Wang H, Pfeifer PE On the equivalence of some medical cost estimators with censored data Stat Med 2007; 26(24): 4520–30 Zhao H, Zuo C, Chen S, Bang H Nonparametric inference for median costs with censored data Biometrics 2012; 68(3): 717–25 Statistics and Epidemiology Economic evaluation has become an essential component of clinical trial design to show that new treatments and technologies offer value to payers in various healthcare systems Although many books exist that address the theoretical or practical aspects of cost-effectiveness analysis, this book differentiates itself from the competition by detailing how to apply health economic evaluation techniques in a clinical trial context, from both academic and pharmaceutical/commercial perspectives It also includes a special chapter for clinical trials in cancer Design & Analysis of Clinical Trials for Economic Evaluation & Reimbursement is not just about performing cost-effectiveness analyses It also emphasizes the strategic importance of economic evaluation and offers guidance and advice on the complex factors at play before, during, and after an economic evaluation Filled with detailed examples, the book bridges the gap between applications of economic evaluation in industry (mainly pharmaceutical) and what students may learn in university courses It provides readers with access to SAS and STATA code In addition, Windows-based software for sample size and value of information analysis is available free of charge—making it a valuable resource for students considering a career in this field or for those who simply wish to know more about applying economic evaluation techniques The book includes coverage of trial design, case report form design, quality of life measures, sample sizes, submissions to regulatory authorities for reimbursement, Markov models, cohort models, and decision trees Examples and case studies are provided at the end of each chapter Presenting first-hand insights into how economic evaluations are performed from a drug development perspective, the book supplies readers with the foundation required to succeed in an environment where clinical trials and cost-effectiveness of new treatments are central It also includes thought-provoking exercises for use in classroom and seminar discussions K14568 w w w c rc p r e s s c o m ... health economics (strategy and analysis) FIGURE 1.1 Description of a multidisciplinary team for designing a clinical trial with a health economic component 4 Design & Analysis of Clinical Trials for. .. applying economic evaluation techniques Design & Analysis of Clinical Trials for Economic Evaluation & Reimbursement: An Applied Approach Using SAS & STATA is structured to cover many of the topics... Chow and Jen-pei Liu Design and Analysis of Bridging Studies Jen-pei Liu, Shein-Chung Chow, and Chin-Fu Hsiao Design & Analysis of Clinical Trials for Economic Evaluation & Reimbursement : An Applied