Design and Analysis of Clinical Trials for Economic Evaluation and Reimbursement: An Applied Approach Using SAS and STATA

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.-- Provided by publisher Content: Introduction to Economic EvaluationHealth Economics, Pharmacoeconomics, and Economic EvaluationImportant Concepts in Economic EvaluationHealth Economic Evaluation and Drug DevelopmentEfficacy, Effectiveness and EfficiencyWhen Is a Pharmacoeconomic Hypothesis Possible?Exercises Health Economic Evaluation ConceptsIncremental Cost-Effectiveness Ratio (ICER)Incremental INMBThe Concept of DominanceTypes of Economic EvaluationStatistical versus Health Economic ModelsExercises Appendix SAS/STATA CodeDesigning Cost-Effectiveness into a Clinical TrialReasons for Collecting Economic Data in a Clinical TrialPlanning a Health Economic Evaluation in a Clinical TrialClinical Trial Design Issues in an Economic EvaluationIntegrating Economic Evaluation in a Clinical Trial: ConsiderationsCRF Design and Data Management IssuesCase Study of a Lung Cancer Trial with an EconomicEvaluationExercises Appendix: SAS/STATAAnalysing Cost Data Collected in a Clinical TrialCollecting and Measuring Costs for the Case Report FormTypes of CostsOther Concepts in Costs: Time Horizon and DiscountingCRFs for Collecting Resource Use Data in Clinical TrialsStatistical Modelling of Cost DataUsing Generalised Linear Models to Analyse Cost DataModels for Skewed Distributions Outside the GLM Family of DistributionsSummary of Modelling ApproachesHandling Censored and Missing CostsStrategies for Avoiding Missing Resource DataStrategies for Analysing Cost Data When Data Are Missing or CensoredImputation MethodsCensored Cost DataMethod of Lin et al. (1997)Summary and ConclusionExercises Appendix: SAS/STATA CodeQuality of Life in Economic EvaluationQuality of Life in Clinical Trials versus Quality of Life for Economic EvaluationDisease-Specific and Generic Measures of HRQoLHRQoL Instruments Used for the Purposes of Economic EvaluationWhen HRQoL Data Have Not Been Collected in a Clinical TrialHRQoL Metrics for Use in Economic EvaluationsAre Utility Measures Sensitive Enough for Detecting Treatment Differences?Exercises Appendix 5A SAS/STATA CodeTechnical Appendix: Beta Binomial Technical DetailsTechnical Appendix: Technical Summary of the GLMModelling in Economic EvaluationIntroduction to Modelling: Statistical versus Economic ModellingDecision Tree ModelsMarkov Modelling/Cohort SimulationAnalysis of Patient-Level DataPatient-Level SimulationOther Issues in ModellingExercises Appendix: SAS/STATA CodeSensitivity AnalysesIntroduction to Sensitivity AnalysisOne-Way Sensitivity AnalysisTwo-Way Sensitivity AnalysisPSABayesian Sensitivity AnalysesIssues in Interpreting and Reporting Results from Sensitivity AnalysisExercises Appendix: SAS/STATA CodeSample Size and Value of Information for Cost-Effectiveness TrialsIntroductionSample Sizes for Cost-EffectivenessSample Size Methods for EfficacySample Size Formulae for Cost-Effectiveness: ExamplesFactors Affecting Sample SizesThe Minimum Sample Size to Establish Cost-EffectivenessBayesian Sample Size ApproachThe Normality AssumptionObtaining the Necessary Data and Tools for CalculatingSample SizeValue of InformationExercises for Chapter 8Appendix 8A SAS/STATA CodeTechnical Appendix 8B Derivation of Sample Size FormulaTechnical Appendix 8C Comparison with Briggs and Tambour's (2001) Approach Mixed Treatment Comparisons, Evidence SynthesisIntroductionMTCsMeta-AnalysisExercises Appendix: SAS/STATA CodeCost-Effectiveness Analyses of Cancer TrialsIntroductionModelling Patient-Level Data from Cancer Trials for Cost-EffectivenessFlexible Parametric Survival ModelsModelling Survival Data Using a Flexible Parametric Model Cost-Effectiveness of Lenalidomide Transition Probabilities and Survival RatesHandling Crossover (Treatment Switching) in Cancer TrialsLandmark Analysis and Presenting Survival Data by Tumour ResponseExercises Appendix: SAS/STATA CodeThe Reimbursement EnvironmentRegulatory Requirements for Clinical Efficacy versus Payer Requirements for ValueReimbursement and Payer Evidence Requirements across Different CountriesMarket Access and StrategyValue-Based PricingSubmissions for Payer EvidenceFurther Areas for ResearchExercises ReferencesBibliographyIndex Front Cover -- Contents -- Preface -- Author -- Acronyms -- Chapter 1: Introduction to Economic Evaluation -- Chapter 2: Health Economic Evaluation Concepts -- Chapter 3: Designing Cost-Effectiveness into a Clinical Trial -- Chapter 4: Analysing Cost Data Collected in a Clinical Trial -- Chapter 5: Quality of Life in Economic Evaluation -- Chapter 6: Modelling in Economic Evaluation -- Chapter 7: Sensitivity Analyses -- Chapter 8: Sample Size and Value of Information for Cost-Effectiveness Trials -- Chapter 9: Mixed Treatment Comparisons, Evidence Synthesis -- Chapter 10: Cost-Effectiveness Analyses of Cancer Trials -- Chapter 11: The Reimbursement Environment -- References -- Bibliography -- Back Cover