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 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 storage or retrieval system, without written permission from the publishers For permission to photocopy or use material electronically from this work, please access www.copyright 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 provides licenses and registration for a variety of users For organizations that have been granted a photocopy 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? J Clin Oncol 2003; 21(24): 4474–5 Gray AM, Clarke PM, Wolstenholme JL, Wordsworth S Applied Methods of CostEffectiveness in Healthcare Oxford: Oxford University Press, 2011 Gregorio GV, Dans LF, Cordero CP, Panelo CA Zinc supplementation reduced cost and duration of acute diarrhea in children J Clin Epidemiol 2007; 60(6): 560–6 Grieve R, Nixon R, Thompson SG, Normand C Using multilevel models for assessing the variability of multinational resource use and cost data Health Econ 2005; 14(2): 185–96 Grieve R, Nixon R, Thompson SG, Cairns J Multilevel models for estimating incremental net benefits in multinational studies Health Econ 2007; 16(8): 815–26 Grootendorst PV A comparison of alternative models of prescription drug utilization Health Econ 1995; 4(3): 183–98 Harper R, Brazier JE, Waterhouse JC, Walters SJ, Jones NM, Howard P Comparison of outcome measures for patients with chronic obstructive pulmonary disease (COPD) in an outpatient setting Thorax 1997; 52(10): 879–87 Haycox A, Drummond M, Walley T Pharmacoeconomics: Integrating economic evaluation into clinical trials Br J Clin Pharmacol 1997; 43(6): 559–62 Hedges LV, Olkin I Statistical Method for Meta Analysis New York: Academic Press, 1985 Herbst RS, Prager D, Hermann R, Fehrenbacher L, Johnson BE, Sandler A, Kris MG, et al TRIBUTE Investigator Group TRIBUTE: A phase III trial of erlotinib hydrochloride (OSI-774) combined with carboplatin and paclitaxel chemotherapy in advanced non-small-cell lung cancer J Clin Oncol 2005; 23(25): 5892–9 Hoaglin DC, Hawkins N, Jansen JP, Scott DA, Itzler R, Cappelleri JC, Boersma C, et al Conducting indirect-treatment-comparison and network-meta-analysis studies: Report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices: part Value Health 2011; 14(4): 429–37 Hornberger J, Eghtesady P The cost-benefit of a randomized trial to a health care organization Control Clin Trials 1998; 19(2): 198–211 Review Huang Y Cost analysis with censored data Med Care 2009; 47(7 Suppl 1): S115–19 Hughes DA, Bagust A, Haycox A, Walley T The impact of non-compliance on the cost-effectiveness of pharmaceuticals: A review of the literature Health Econ 2001; 10(7): 601–15 Hui-Min W, Ming-Fang Y, Chen TH SAS macro program for non-homogeneous Markov process in modeling multi-state disease progression Computer methods Programs Biomed 2004; 75(2): 95–105 Idiok-Akpan PA, Nonye AA Perceptions of burden of caregiving by informal caregivers of cancer patients attending University of Calabar Teaching Hospital, Calabar, Nigeria Pan Afr Med J 2014; 18: 159 (using GLOBOCAN, Cancer Fact Sheet- Cancer Incidence and Mortality Worldwide International Agency for Cancer Research, 2008 http://www.iarc.fr/en/media-centre/iarcnews/2010/globocan2008.php) Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) (Institute for Quality and Efficiency in Health Care) General methods; Version 3.0 of 27 May 2008 References 291 Ishak KJ, Caro JJ, Drayson MT, Dimopoulos M, Weber D, Augustson B, Child JA, et al Adjusting for patient crossover in clinical trials using external data: A case study of lenalidomide for advanced multiple myeloma Value Health 2011; 14(5): 672–78 Jansen JP, Fleurence R, Devine B, Itzler R, Barrett A, Hawkins N, Lee K, Boersma C, Annemans L, Cappelleri JC Interpreting indirect treatment comparisons and network meta-analysis for health-care decision making: Report of the ISPOR Task Force on Indirect Treatment Comparisons Good Research Practices: Part Value Health 2011; 14(4): 417–28 Keus F, de Jonge T, Gooszen HG, Buskens E, van Laarhoven CJ Cost-minimization analysis in a blind randomized trial on small-incision versus laparoscopic cholecystectomy from a societal perspective: Sick leave outweighs efforts in hospital savings Trials 2009; 10: 80 Kharroubi SA, O’Hagan A, Brazier JE A comparison of United States and United Kingdom EQ-5D health states valuations using a nonparametric Bayesian method Stat Med 2010; 29(15): 1622–34 Khan I Probabilistic sensitivity analyses for clinical trials of cost-effectiveness using the method of copulas: A comparison of simulation methods SCT Meeting Abstracts Society for Clinical Trials 2014 Khan I Interpreting small effects from the EORTC-QLQC-30 2015 Health Qual Life Outcomes (in press) Khan I SCET-VA® Version released May 2015; Sample size software for cost-effectiveness and value of information analysis Available from author on request at https://www.researchgate.net/profile/Iftekhar_Khan5 Khan I, Morris S A non-linear beta-binomial regression model for mapping EORTC QLQ- C30 to the EQ-5D-3L in lung cancer patients: A comparison with existing approaches Health Qual Life Outcomes 2014; 12: 163 Khan I, Morris S, Hackshaw A, Lee SM Cost effectiveness of first-line erlotinib in patients with advanced non-small-cell lung cancer unsuitable for chemotherapy BMJ [Open] 2015; 5(7): e006733 Khan I, Pashayan N, Matata B, Morris S, Maguire J Sensitivity and responsiveness of EQ-5D-5L compared with EQ-5D-3L and EORTC-QLQC-30 in non-small cell lung cancer patients Health Qual Life Outcomes (in press) Kobelt G Health Economics An Introduction to Economic Evaluation, 2nd edn London: Office of Health Economics, 1996 Kruger CJC Constrained cubic spline interpolation for chemical engineering purposes, 2003 Available at: http://www.korf.co.uk/spline.pdf Lambert PC, Billingham LJ, Cooper NJ, Sutton AJ, Abrams KR Estimating the costeffectiveness of an intervention in a clinical trial when partial cost information is available: A Bayesian approach Health Econ 2008; 17(1): 67–81 Laska EM, Meisner M, Siegel C Power and sample size in cost-effectiveness analysis Med Decis Making 1999; 19(3): 339–43 Leal J, Gray AM, Clarke PM Development of life-expectancy tables for people with type diabetes Eur Heart J 2009; 30(7): 834–9 Lee SM, Khan I, Upadhyay S, Lewanski C, Falk S, Skailes G, Marshall E, et al Firstline erlotinib in patients with advanced non-small-cell lung cancer unsuitable for chemotherapy (TOPICAL): A double-blind, placebo-controlled, phase trial Lancet Oncol 2012; 13(11): 1161–70 292 References Lewis G, Peake M, Aultman R, Gyldmark M, Morlotti L, Creeden J, de la Orden M Cost-effectiveness of erlotinib versus docetaxel for second-line treatment of advanced non-small-cell lung cancer in the United Kingdom J Int Med Res 2010; 38(1): 9–21 Lin DY Proportional means regression for censored medical costs Biometrics 2000; 56(3): 775–8 Lin DY, Feuer EJ, Etzioni R, Wax Y Estimating medical costs from incomplete followup data Biometrics 1997; 53(2): 419–34 Liu L, Strawderman RL, Cowen ME, Shih YC A flexible two-part random effects model for correlated medical costs J Health Econ 2010; 29(1): 110–23 Machin D, Campbell MJ, Tan SB, Tan SH Sample Size Tables for Clinical Studies, 3rd edn Oxford: John Wiley, 2009 Maguire J, Khan I, McMenemin R, O’Rourke N, McNee S, Kelly V, Peedell C, Snee M SOCCAR: A randomised phase II trial comparing sequential versus concurrent chemotherapy and radical hypofractionated radiotherapy in patients with inoperable stage III non-small cell lung cancer and good performance status Eur J Cancer 2014; 50(17): 2939–49 Maiwenn J, Van Hout B A Bayesian approach to economic analyses of clinical trials: The case of stenting versus balloon angioplasty Health Econ 2000; 9(7): 599–609 Manca A, Rice N, Sculpher MJ, Briggs AH Assessing generalisability by location in trial-based cost-effectiveness analysis: The use of multilevel models Health Econ 2005; 14(5): 471–85 Erratum in Health Econ 2005; 14(5): 486 Manca A, Lambert PC, Sculpher M, Rice N Cost-effectiveness analysis using data from multinational trials: The use of bivariate hierarchical modeling Med Decis Making 2007; 27(4): 471–90 Mann R, Brazier J, Tsuchiya A A comparison of patient and general population weightings of EQ-5D dimensions Health Econ 2009; 18(3): 363–72 Manning WG, Mullahy J Estimating log models: To transform or not to transform? J. Health Econ 2001; 20(4): 461–94 McKenzie L, van der Pol M Mapping the EORTC QLQ C-30 onto the EQ-5D instrument: The potential to estimate QALYs without generic preference data Value Health 2009; 12(1): 167–71 Mihaylova B, Briggs A, O’Hagan A, Thompson SG Review of statistical methods for analysing healthcare resources and costs Health Econ 2011; 20(8): 897–916 Morden JP, Lambert PC, Latimer NR, Abrams KR, Wailoo AJ Assessing methods for dealing with treatment switching in randomised controlled trials: A simulation study BMC Med Res Methodol 2011; 11: Nikolakopoulou A, Mavridis D, Salanti G Using conditional power of network metaanalysis (NMA) to inform the design of future clinical trials Biometrical J 2014; 56(6): 973–90 NICE Guide to the methods of technology appraisal 2013; published April 2013 http://publications.nice.org.uk/pmg9 Nixon RM, Thompson SG Parametric modelling of cost data in medical studies Stat Med 2004; 23: 1311–31 Noble S, Hollingworth W, Tilling K Missing data in trial-based cost-effectiveness analysis: The current state of play Health Econ 2012; 21: 187–200 O’Connor RD, O’Donnell JC, Pinto LA, Wiener DJ, Legorreta AP Two-year retrospective economic evaluation of three dual-controller therapies used in the treatment of asthma Chest 2002; 121(4): 1028–35 References 293 O’Hagan A, Stevens JW Bayesian assessment of sample size for clinical trials of costeffectiveness Med Decis Making 2001; 21: 219–30 O’Hagan A, Stevens JW Bayesian methods for design and analysis of cost-effectiveness trials in the evaluation of health care technologies Stat Meth Med Res 2002; 11: 469–90 Osoba D, Rodrigues G, Myles J, Zee B, Pater J Interpreting the significance of changes in health-related quality-of-life scores J Clin Oncol 1998; 16(1): 139–44 O’Sullivan AK, Thompson D, Drummond MF Collection of health-economic data alongside clinical trials: Is there a future for piggyback evaluations? Value Health 2005; 8(1): 67–79 Petrou S, Gray A Economic evaluation alongside randomised controlled trials: Design, conduct, analysis, and reporting BMJ 2011a; 342: d1548 Petrou S, Gray A Economic evaluation using decision analytical modelling: Design, conduct, analysis, and reporting BMJ 2011b; 342: d1766 Pocock SJ Clinical Trials: A Practical Approach Chichester: John Wiley, 1983 Popat R, Khan I, Dickson J, Cheesman S, Smith D, D’Sa S, Rabin N, Yong K An alternative dosing strategy of lenalidomide for patients with relapsed multiple myeloma Br J Haematol 2015; 168(1): 148–51 Postma MJ, de Vries R, Welte R, Edmunds WJ Health economic methodology illustrated with recent work on Chlamydia screening: The concept of extended dominance Sex Transm Infect 2008: 84(2): 152–4 Ramsey S, Willke R, Briggs A, Brown R, Buxton M, Chawla A, Cook J, Glick H, Liljas B, Petitti D, Reed S Good research practices for cost-effectiveness analysis alongside clinical trials: The ISPOR RCT-CEA Task Force report Value Health 2005; 8(5): 521–33 Raikou M, McGuire A Estimating medical care costs under conditions of censoring J Health Econ 2004; 23(3): 443–70 Ramsey SD, Willke RJ, Glick H, Reed SD, Augustovski F, Jonsson B, Briggs A, Sullivan SD Cost-effectiveness analysis alongside clinical trials II – An ISPOR Good Research Practices Task Force report Value Health 2015; 18(2): 161–72 Rascati KL Essentials of Pharmacoeconomics, 2nd edn Baltimore: Lippincott Williams & Wilkins, 2013 Regan J, Yarnold J, Jones PW, Cooke NT Palliation and life quality in lung cancer; how good are clinicians at judging treatment outcome? Br J Cancer 1991; 64(2): 396–400 Rittenhouse BE, Dulisse B, Stinnett AA At what price significance? The effect of price estimates on statistical inference in economic evaluation Health Econ 1999; 8(3): 213–19 Robins JM, Finkelstein DM Correcting for noncompliance and dependent censoring in an AIDS Clinical Trial with inverse probability of censoring weighted (IPCW) log-rank tests Biometrics 2000; 56(3): 779–88 Robins JM, Hernán MA, Brumback B Marginal structural models and causal inference in epidemiology Epidemiology 2000; 11(5): 550–60 Robinson R Economic evaluation and health care What does it mean? BMJ 1993; 307(6905): 670–3 Rowen D, Brazier J, Young T, Gaugris S, Craig BM, King MT, Velikova G Deriving a preference-based measure for cancer using the EORTC QLQ-C30 Value Health 2011; 14(5): 721–31 Royston P, Lambert PC Flexible Parametric Survival Analysis using STATA: Beyond the Cox Model Texas: Stata Press, 2011 294 References Royston P, Parmar MK Flexible parametric proportional-hazards and proportionalodds models for censored survival data, with application to prognostic modelling and estimation of treatment effects Stat Med 2002; 21(15): 2175–97 Rubin DB Multiple Imputation for Nonresponse in Surveys New York: John Wiley, 1987 Rutten-van Mölken MP, van Doorslaer EK, van Vliet RC Statistical analysis of cost outcomes in a randomized controlled clinical trial Health Econ 1994; 3(5): 333–45 Sakia RM The Box-Cox transformation technique: A review J Roy Stat Soc Series D (The Statistician) 1992; 41(2): 169–78 Santerre RE, Neun SP Health Economics: Theory, Insights and Industry Studies, 5th edn Orlando, FL: Cengage Learning, 2009 Sarker SJ, Whitehead A Factors affecting the sample size of cost-effectiveness trials Technical Report, MPS Research Unit, Reading University, 2004 Sarker SJ, Whitehead A, Khan I A C++ program to calculate sample sizes for costeffectiveness trials in a Bayesian framework Comput Methods Programs Biomed 2013; 110(3): 471–89 Sculpher MJ, Claxton K, Drummond M, McCabe C Whither trial-based economic evaluation for health care decision making? Health Econ 2006; 15(7): 677–87 Senn S Statistical Issues in Drug Development New York: John Wiley, 1997 Sharples LD, Edmunds J, Bilton D, Hollingworth W, Caine N, Keogan M, Exley A A randomised controlled crossover trial of nurse practitioner versus doctor led outpatient care in a bronchiectasis clinic Thorax 2002; 57(8): 661–6 Shepherd FA, Pereira JR, Ciuleanu T, Tan EH, Hirsh V, Thongprasert S, Campos D, et al National Cancer Institute of Canada Clinical Trials Group Erlotinib in previously treated non-small-cell lung cancer N Engl J Med 2005; 353(2): 123–32 Siani C, Moatti J-P The handling of uncertainty in economic evaluations of health care strategies Rev Epidemiol Sante Publique 2003; 51(2): 255–76 Simons CL, Rivero-Arias O, Yu LM, Simon J Multiple imputation to deal with missing EQ-5D-3L data: Should we impute individual domains or the actual index? Qual Life Res 2015; 24(4): 805–15 Sleeper LA, Harrington DP Regression splines in the Cox model with application to covariate effects in liver disease J Am Stat Assoc 1990; 85: 941–9 Slevin ML, Plant H, Lynch D, Drinkwater J, Gregory WM Who should measure quality of life, the doctor or the patient? Br J Cancer 1988; 57(1): 109–12 Soares MO, Iglesias CP, Bland JM, Cullum N, Dumville JC, Nelson EA, Torgerson DJ, Worthy G: VenUS II team Cost effectiveness analysis of larval therapy for leg ulcers BMJ 2009; 338: b825 Spiegelhalter DJ, Abrams KR, Myles JP Bayesian Approaches to Clinical Trials and Health Care Evaluation Chichester: John Wiley, 2004 Stadtmauer EA, Weber DM, Niesvizky R, Belch A, Prince MH, San Miguel JF, Facon T, et al Lenalidomide in combination with dexamethasone at first relapse in comparison with its use as later salvage therapy in relapsed or refractory multiple myeloma Eur J Haematol 2009; 82(6): 426–32 Stinnett AA, Mullahy J Net health benefits: A new framework for the analysis of uncertainty in cost-effectiveness analysis Med Decis Making 1998; 18(Suppl.): S68–80 Stokes ME, Davis CS Categorical Data Analysis Using the SAS System Cary: SAS Institute Publication, 2009 References 295 Thatcher N, Chang A, Parikh P, Pereira JR, Ciuleano T, Pawel JV, Thongprasert S, et al Geftinib plus best supportive care in previously treated patients with refractory advanced non-small-cell lung cancer: Results from a randomised, placebo-controlled, multicentre study (Iressa Survival Evaluation in Lung Cancer) Lancet 2005; 366(9496): 1527–37 Thompson SG, Nixon RM, Grieve R Addressing the issues that arise in analysing multicentre cost data, with application to a multinational study J Health Econ 2006; 25(6): 1015–28 Vale CD, Maurelli VA Simulating multivariate nonnormal distributions Psychometrika 1983; 48: 465–71 Welton N, Ades AE Research decisions in the face of heterogeneity: What can a new study tell us? Health Econ 2012; 21(10): 1196–200 Whitehead J, Valdés-Márquez E, Johnson P, Graham G Bayesian sample size for exploratory clinical trials incorporating historical data Stat Med 2008; 27: 2307–27 Wijeysundera HC, Wang X, Tomlinson G, Ko DT, Krahn MD Techniques for estimating health care costs with censored data: An overview for the health services researcher Clinicoecon Outcomes Res 2012; 4: 145–55 Willan AR, Lin DY, Cook RJ, Chen EB Using inverse-weighting in cost-effectiveness analysis with censored data Stat Methods Med Res 2002; 11(6): 539–51 Willke RJ, Glick HA, Polsky D, Schulman K Estimating country-specific costeffectiveness from multinational clinical trials Health Econ 1998; 7(6): 481–93 Zhang L, Ma S, Song X, Han B, Cheng Y, Huang C, Yang S, Liu X, Liu Y, Lu S, Wang J, Zhang S, Zhou C, Zhang X, Hayashi N, Wang M; INFORM investigators Gefitinib versus placebo as maintenance therapy in patients with locally advanced or metastatic non-small-cell lung cancer (INFORM; C-TONG 0804): A multicentre, double-blind randomised phase trial Lancet Oncol 2012; 13(5): 466–75 Bibliography Al MJ, Van Hout BA A Bayesian approach to economic analyses of clinical trials: The case of stenting versus balloon angioplasty Health Econ 2000; 9(7): 599–609 Arnold RJG Pharmacoeconomics: From Theory to Practice Boca Raton: CRC Press, 2010 Arnold RG, Kotsanos JG Panel: Methodological issues in conducting pharmacoeconomic evaluations – retrospective and claims database studies Value Health 1999; 2(2): 82–7 Askew RL, Swartz RJ, Xing Y, Cantor SB, Ross MI, Gershenwald JE, Palmer JL, Lee JE, Cormier JN Mapping FACT-melanoma quality-of-life scores to EQ-5D health utility weights Value Health 2011; 14(6): 900–6 Bennett CL, Westerman IL Economic analysis during phase III clinical trials: Who, what, when, where, and why? Oncology (Williston Park) 1995; 9(11 Suppl.): 169–75 Berlin JA, Cepeda MS Some methodological points to consider when performing systematic reviews in comparative effectiveness research Clin Trials 2012; 9(1): 27–34 Bleichrodt H, Filko M New tests of QALYs when health varies over time J Health Econ 2008; 27(5): 1237–49 Blough DK, Ramsey S, Sullivan SD, Yusen R Nett Research Group The impact of using different imputation methods for missing quality of life scores on the estimation of the cost-effectiveness of lung-volume-reduction surgery Health Econ 2009; 18(1): 91–101 Brazier J, Ratcliffe J, Salomon JA, Tsuchiya A Measuring and Valuing Health Benefits for Economic Evaluation Oxford: Oxford University Press, 2007 Brennan A, Chick SE, Davies R A taxonomy of model structures for economic evaluation of health technologies Health Econ 2006; 15(12): 1295–1310 Briggs A, Fenn P Confidence intervals or surfaces? Uncertainty on the cost-effectiveness plane Health Econ 1998; 7(8): 723–40 Briggs A, Clark T, Wolstenholme J, Clarke P Missing … presumed at random: Costanalysis of incomplete data Health Econ 2003; 12: 377–92 Bryan S, Williams I, McIver S Seeing the NICE side of cost-effectiveness analysis: A qualitative investigation of the use of CEA in NICE technology appraisals Health Econ 2007; 16(2): 179–93 Buxton MJ, Drummond MF, Van Hout BA, Prince RL, Sheldon TA, Szucs T, Vray M Modelling in economic evaluation: An unavoidable fact of life Health Econ 1997; 6(3): 217–27 Cancer Research UK GEDDIS Trial unpublished results: http://www.isrctn.com/ ISRCTN07742377 Cantoni E, Ronchetti E A robust approach for skewed and heavy-tailed outcomes in the analysis of health care expenditures J Health Econ 2006; 25(2): 198–213 Claxton K Heterogeneity in cost-effectiveness of medical interventions: The Cochrane Review 2008; challenges of matching patients to appropriate care; 3 November 2011; 14th Annual ISPOR Congress, 1–8 November 2011 297 298 Bibliography Contreras-Hernández I, Mould-Quevedo JF, Torres-González R, Goycochea-Robles MV, Pacheco-Domínguez RL, Sánchez-García S, Mejía-Aranguré JM, GardoEspinosa J Cost-effectiveness analysis for joint pain treatment in patients with osteoarthritis treated at the Instituto Mexicano del Seguro Social (IMSS): Comparison of nonsteroidal anti-inflammatory drugs (NSAIDs) vs cyclooxygenase-2 selective inhibitors Cost Eff Resour Alloc 2008; 12(6): 21 Cooper NJ, Sutton AJ, Abrams KR, Turner D, Wailoo A Comprehensive decision analytical modelling in economic evaluation: A Bayesian approach Health Econ 2004; 13(3): 203–26 Coyle D, Drummond MF Analyzing differences in the costs of treatment across centers within economic evaluations Int J Technol Assess Health Care 2001; 17(2): 155–63 Craig BA, Sendi PP Estimation of the transition matrix of a discrete-time Markov chain Health Econ 2002; 11(1): 33–42 Craig MB The duration effect: A link between TTO and VAS values Health Econ 2009; 18(2): 217–25 Cramer JA, Spilker B Quality of Life and Pharmacoeconomics Philadelphia: LippincottRaven, 1998 Díaz-Ordaz K, Kenward MG, Grieve R Handling missing values in cost effectiveness analyses that use data from cluster randomized trials J Roy Stat Soc Ser A (Statistics in Society) 2014; 177(2): 457–74 Dreyer NA, Schneeweiss S, McNeil BJ, Berger ML, Walker AM, Ollendorf DA, Gliklich RE GRACE principles: Recognizing high-quality observational studies of comparative effectiveness GRACE Initiative Am J Manag Care 2010; 16(6): 467–71 Eckermann S, Willan AR Globally optimal trial design for local decision making Health Econ 2009; 18(2): 203–16 European Medicines Agency Guideline on the missing data; EMA/CPMP/ EWP/1776/99 Rev European Medicines Agency; Oncology Working Party Reflection Paper on the use of patient reported outcome (PRO) measures in oncology studies EMA/ CHMP/292464/2014 EMA, 2014 Evidence Review Group The clinical- and cost-effectiveness of lenalidomide for multiple myeloma in people who have received at least one prior therapy: An evidence review of the submission from CELGENE; PenTAG on behalf of NICE, September 2008 Feenstra TL, van Baal PH, Gandjour A, Brouwer WB Future costs in economic evaluation A comment on Lee J Health Econ 2008; 27(6): 1645–9; discussion 1650–1 Fox-Rushby J, Cairns J eds., Economic Evaluation Maidenhead: Open University Press, 2005 Gardiner JC Modeling heavy-tailed distributions in healthcare utilization by parametric and Bayesian methods SAS Global Forum, 2012 Graves N, Walker D, Raine R, Hutchings A, Roberts JA Cost data for individual patients included in clinical studies: No amount of statistical analysis can compensate for inadequate costing methods Health Econ 2002; 11(8): 735–9 Review Grieve R, Cairns J, Thompson SG Improving costing methods in multicentre economic evaluation: The use of multiple imputation for unit costs Health Econ 2010; 19(8): 939–54 Bibliography 299 Hardy KJ, Szczepura A, Davies R, Bradbury A, Stallard N, Gossain S, Walley P, Hawkey PM A study of the efficacy and cost-effectiveness of MRSA screening and monitoring on surgical wards using a new, rapid molecular test (EMMS) BMC Health Serv Res 2007; 7: 160 Hawkins N, Scott DA Reimbursement and value-based pricing: Stratified costeffectiveness analysis may not be the last word Health Econ 2011; 20(6): 688–98 Heitjan DF, Li H Bayesian estimation of cost-effectiveness: An importance-sampling approach Health Econ 2004; 13(2): 191–8 Hlatky MA Economic endpoints in clinical trials Epidemiol Rev 2002; 24(1): 80–4 Jackson CH, Thompson SG, Sharples LD Accounting for uncertainty in health economic decision models by using model averaging J R Stat Soc Ser A Stat Soc 2009; 172(2): 383–404 Jackson CH, Bojke L, Thompson SG, Claxton K, Sharples LD A framework for addressing structural uncertainty in decision models Med Decis Making 2011; 31(4): 662–74 Jonas ED, Wilkins MT, Bangdiwala, Bann CM, Morgan LC, Thaler KJ, Amick HR, Gartlehner G Findings of a Bayesian mixed treatment comparison meta analyses: Comparison and exploration using real-world trial data and simulation Agency for Healthcare Research and Quality; February 2013 Jönsson L, Sandin R, Ekman M, Ramsberg J, Charbonneau C, Huang X, Jönsson B, Weinstein MC, Drummond M Analyzing overall survival in randomized controlled trials with crossover and implications for economic evaluation Value Health 2014; 17(6): 707–13 Kaambwa B, Billingham L, Bryan S Mapping utility scores from the Barthel index Eur J Health Econ 2013; 14(2): 231–41 Karnon J, Goyder E, Tappenden P, McPhie S, Towers I, Brazier J, Madan J A review and critique of modelling in prioritising and designing screening programmes Health Technol Assess 2007; 11(52): iii–iv, ix–xi, 1–145 Klein JP, Moeschberger ML Survival Analysis: Techniques for Censored and Truncated Data (Statistics for Biology and Health), 3rd edn Springer, 2005 Kreif N, Grieve R, Radice R, Sadique Z, Ramsahai R, Sekhon JS Methods for estimating subgroup effects in cost-effectiveness analyses that use observational data Med Decis Making 2012; 32(6): 750–63 Kunz R, Vist G, Oxman AD Randomisation to protect against selection bias in healthcare trials Cochrane Database Syst Rev 2007; (2): MR000012 Latimer N, Abrams KR NICE DSU Technical Support Document 14: Survival Analysis for Economic Evaluations alongside Clinical Trials – Extrapolation with Patient Level Data; Report by the Decision Support Unit, June 2011 Longworth L, Rowen D NICE DSU Technical Support Document 10: The Use of Mapping Methods to Estimate Health Utility State Values; Report by the Decision Support Unit, April 2011 Manca A, Sculpher MJ, Goeree R The analysis of multinational cost-effectiveness data for reimbursement decisions Pharmacoeconomics 2010; 28(12): 1079–96 Marsh K, Phillips CJ, Fordham R, Bertranou E, Hale J Estimating cost-effectiveness in public health: A summary of modelling and valuation methods Health Econ Rev 2012; 2(1): 17 Muening P Cost-Effectiveness Analysis in Health A Practical Approach, 2nd edn New York: John Wiley, 2008 300 Bibliography Nixon RM, Thompson SG Methods for incorporating covariate adjustment, subgroup analysis and between-centre differences into cost-effectiveness evaluations Health Econ 2005; 14(12): 1217–29 Oostenbrink JB, Al MJ The analysis of incomplete cost data due to dropout Health Econ 2005; 14(8): 763–76 Papaioannou D, Brazier J, Paisley S NICE DSU Technical Support Document 9: The Identification Review and Synthesis of Health State Utility Values from the Literature; Report by the Decision Support Unit, October 2010 Parker M, Haycox A, Graves J Estimating the relationship between preference-based generic utility instruments and disease-specific quality-of-life measures in severe chronic constipation: Challenges in practice Pharmacoeconomics 2011; 29(8): 719–30 Philips Z, Ginnelly L, Sculpher M, Claxton K, Golder S, Riemsma R, Woolacoot N, Glanville J Review of guidelines for good practice in decision-analytic modelling in health technology assessment Health Technol Assess 2004; 8(36): iii–iv, ix–xi, 1–158 Pohlmeier W, Ulrich V An econometric model of the two-part decision making process in the demand for health care J Hum Resour 1995; 30(2): 339–61 Prieto L, Sacristán JA Problems and solutions in calculating quality-adjusted life years (QALYs) Health Qual Life Outcomes 2003; 1: 80 Ridderstråle M, Jensen MM, Gjesing RP, Niskanen L Cost-effectiveness of insulin detemir compared with NPH insulin in people with type diabetes in Denmark, Finland, Norway, and Sweden J Med Econ 2013; 16(4): 468–78 Rychlik R Strategies in Pharmacoeconomics and Outcomes Research New York: Pharmaceuticals Products Press, 2002 Sassi F Calculating QALYs, comparing QALY and DALY calculations Health Policy Plan 2006; 21(5): 402–8 Schmitz S, Adams R, Walsh CD, Barry M, FitzGerald O A mixed treatment comparison of the efficacy of anti-TNF agents in rheumatoid arthritis for methotrexate non-responders demonstrates differences between treatments: A Bayesian approach Ann Rheum Dis 2012; 71(2): 225–30 Scott DA, Boye KS, Timlin L, Clark JF, Best JH A network meta-analysis to compare glycaemic control in patients with type diabetes treated with exenatide once weekly or liraglutide once daily in comparison with insulin glargine, exenatide twice daily or placebo Diabetes Obes Metab 2013; 15(3): 213–23 Sculpher M Reflecting heterogeneity in patient benefits: The role of subgroup analysis with comparative effectiveness Value Health 2010; 13(Suppl 1): S18–S21 Severens JL, Brunenberg DE, Fenwick EA, O’Brien B, Joore MA Cost-effectiveness acceptability curves and a reluctance to lose Pharmacoeconomics 2005; 23(12): 1207–14 Review Simes RJ, Coates AS Patient preferences for adjuvant chemotherapy of early breast cancer: How much benefit is needed? J Natl Cancer Inst Monogr 2001; (30): 146–52 Singer DJ Using SAS PROC MIXED to fit multilevel models, hierarchical models, and individual growth models J Educ Behav Stat 1988; (4): 323–55 Soto J Health economic evaluations using decision analytic modeling Int J Technol Assess Health Care 2002; 18(1): 94–111 Bibliography 301 Sutton A, Ades AE, Cooper N, Abrams K Use of indirect and mixed treatment comparisons for technology assessment Pharmacoeconomics 2008; 26(9): 753–67 Review Tambour M, Zethraeus N, Johannesson M A note on confidence intervals in costeffectiveness analysis Int J Technol Assess Health Care 1998; 4: 467–71 Torrance GW Measurement of health state utilities for economic appraisal J Health Econ 1986; 5(1): 1–30 Tsuchiya A The allocation of health care resources: The basic concepts (in Japanese) In Kato H, Kamo N, eds Handbook for Bioethics, Tokyo: Sekai Shiso Sha, 1998 Tsuchiya A, Williams A Welfare economics and economic evaluation In Drummond M, McGuire A, eds., Economic Evaluation in Health Care: Theory and Practice, Chapter New York: Oxford University Press, 2001 Watkins C, Huang X, Latimer N, Tang Y, Wright EJ Adjusting overall survival for treatment switches: Commonly used methods and practical application Pharm Stat 2013; 12(6): 348–57 Willan AR Sample size determination for cost-effectiveness trials Pharmacoeconomics 2011; 29(11): 933–49 Willan AR, Briggs AH Statistical Analysis of Cost-Effectiveness Data Chichester: John Wiley, 2006 Willan AR, Eckermann S Accounting for between-study variation in incremental net benefit in value of information methodology Health Econ 2012; 21(10): 1183–95 Willan AR, Kowgier ME Cost-effectiveness analysis of a multinational RCT with a binary measure of effectiveness and an interacting covariate Health Econ 2008; 17(7): 777–91 Willan AR, O’Brien BJ Health Econ 1996a; 5(4): 297–305 Erratum in Health Econ 1999; 8(6): 559 Willan AR, O’Brien BJ Confidence intervals for cost-effectiveness ratios: An application of Fieller’s theorem Health Econ 1996b; 5: 297–305 Willan AR, Briggs AH, Hoch JS Regression methods for covariate adjustment and subgroup analysis for non-censored cost-effectiveness data Health Econ 2004; 13(5): 461–75 Wordsworth S, Ludbrook A Comparing costing results in across country economic evaluations: The use of technology specific purchasing power parities Health Econ 2005; 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