Design & analysis of clinical trials for economic evaluation & reimbursement : an applied approach using SAS & STATA

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