| Description |
1 online resource (443 pages) |
| Series |
Chapman and Hall/CRC Biostatistics Ser |
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Chapman and Hall/CRC Biostatistics Ser
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| Contents |
Cover -- Half Title -- Series Page -- Title Page -- Copyright Page -- Dedication -- Table of Contents -- Preface -- Acknowledgments -- About the Authors -- Acronyms and Abbreviations -- 1: Introduction to Cancer -- 1.1"Cancer -- 1.2"Epidemiology of Cancer -- 1.2.1"Cancer Trends -- 1.3"Prognostic Factors Associated with Cancer Outcomes -- 1.4"Economic Burden of Cancer -- 1.4.1"Health Expenditure -- 1.4.2"Healthcare Expenditure on Drugs -- 1.5"Treatments for Cancer -- 1.6"Important Economic Concepts for Cost-Effectiveness of Cancer Interventions -- 1.6.1"Economics, Health Economics, Economic Evaluation, and Pharmacoeconomics -- 1.6.1.1"Value -- 1.6.1.2"Allocative Efficiency -- 1.6.1.3"Technical Efficiency -- 1.6.1.4"Opportunity Cost -- 1.6.1.5"Discounting -- 1.6.1.6"The Incremental Cost-Effectiveness Ratio -- 1.6.1.7"The Cost-Effectiveness Plane -- 1.6.1.8"Quality-Adjusted Life-Years (QALY) -- 1.7"Health Economic Evaluation and Cancer Drug Development in Practice -- 1.7.1"The Modern Paradigm -- 1.8"Efficacy versus Effectiveness -- 1.9"Real-World Data -- 1.10"Economic versus Clinical Hypotheses -- 1.11"Summary -- 1.12"Exercises for Chapter 1 -- 2: Important Outcomes for Economic Evaluation in Cancer Studies -- 2.1"Introduction -- 2.2"Important Common, Surrogate, and Novel Cancer Endpoints -- 2.2.1"Overall Survival -- 2.2.1.1"OS and Economic Evaluation -- 2.2.2"Surrogate Endpoints -- 2.3"HTAs with Surrogate Endpoints -- 2.4"Emerging Tumor-Centered Endpoints -- 2.5"Demonstrating Value from Other Cancer Endpoints -- 2.6"Summary -- 2.7"Exercises for Chapter 2 -- 3: Health-Related Quality of Life for Cost-Effectiveness -- 3.1"Health-Related Quality of Life (HRQoL) in Cancer Patients -- 3.1.1"Limitations of Anti-Cancer Treatments -- 3.1.2"Why Collect HRQoL Data? -- 3.1.3"Challenges with HRQoL in Cancer Studies |
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3.2"Measuring Health-Related Quality of Life Outcomes for Common Cancer Types -- 3.2.1"Condition-Specific Measures of HRQoL -- 3.2.2"Common General Condition-Specific Measures of HRQoL in Cancer -- 3.3"Measuring HRQoL for Economic Evaluation -- 3.3.1"EuroQol EQ-5D-3L and 5L -- 3.3.2"EuroQol EQ-5D-5L -- 3.4"Constructing Utilities -- 3.5"Quality-Adjusted Life-Years (QALYs) -- 3.5.1"QALY Calculation in Cancer Trials -- 3.6"Economic Evaluation in the Absence of Utility Data: Mapping and Utility Studies -- 3.7"Sensitivity and Responsiveness of EQ-5D versus QLQ-C30 HRQoL for Detecting Improvement in Cancer Patients -- 3.8#Measuring Post-Progression (PP) Utility: Some Approaches -- Why Is Estimation of Utility between Disease Progression and Death Relevant? -- The Behavior of Utility in Cancer Patients between Progression and Death? -- 3.8.1"Plausible Post-Progression Utility Behavior -- 3.8.2"Non-Linear Models -- 3.9"HRQoL Issues in Health Technology Appraisals of Cancer Drugs -- 3.10"Summary -- 3.11"Exercises for Chapter 3 -- 4: Introductory Statistical Methods for Economic Evaluation in Cancer -- 4.1"Introduction -- 4.2"Uncertainty and Variability -- 4.2.1"Uncertainty -- 4.2.2"Variability -- 4.2.2.1"Hypothesis Testing -- 4.3"Distributions: Cost, Utility, and Survival Data -- 4.4"Important Measures Used in Cancer Trials -- 4.4.1"Time-to-Event Endpoints -- 4.4.2"Median Survival -- 4.4.3"Hazard Rate and Hazard Ratio -- 4.4.4"Hazard Ratio -- 4.4.5"Survival Rates and Proportions -- 4.4.6"Relationship between Hazard Rate and Survival Rate -- 4.4.7"Transition Probability and Matrix -- 4.4.8"Relation between Transition Probability and Survival Rates -- 4.4.9"Proportional Hazards -- 4.4.10"Mean Survival and Restricted Mean -- 4.5"Simulation: Bootstrapping and Monte-Carlo Simulation -- 4.5.1"Simulating Using Monte-Carlo Sampling |
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4.6"Analyzing Data from Cancer Trials -- 4.6.1"Semi-Parametric Methods: The Cox PH Model -- 4.6.1.1"Adjusting for Covariates with the Cox Model -- 4.6.1.2"Using Hazard Ratios to Predict Survival Rates -- 4.6.2"Parametric Methods: Modeling Survival Data for Extrapolation -- 4.6.3"Advanced Modeling Techniques for Survival Data -- 4.6.3.1"Flexible Parametric Survival Models -- 4.6.3.2"Applications in Cancer Surveillance -- 4.7"Issues in Fitting Models -- 4.8"Handling Crossover, Treatment Switching, and Subsequent Anti-Cancer Therapy -- 4.8.1"Introduction to Treatment Switching -- 4.8.2"Types of Switching -- 4.8.3"Implications of Switching -- 4.8.4"Handling Switching in Statistical Analyses -- 4.8.4.1"Intent-to-Treat (ITT) -- 4.8.4.2"Per Protocol Analysis -- 4.8.4.3"IPCW -- 4.8.4.4"RPFSTM -- 4.8.4.5"Two-Stage Adjustment Model -- 4.8.4.6"Other Approaches: Structural Nested Mean Models (SNNM) -- 4.10"Summary -- 4.11"Exercises for Chapter 4 -- 4.9"Data Synthesis and Network Meta-Analyses -- 4.9.1"Mixed Treatment Comparisons -- 4.9.1.1"Direct Comparison -- 4.9.1.2"Indirect Treatment Comparison (ITC) -- 4.9.1.3"Meta-Analysis -- 4.9.1.4"Network of Evidence -- 4.9.2"Assumptions for Carrying Out MTCs -- 4.10"Summary -- 4.11"Exercises for Chapter 4 -- 5: Collecting and Analysis of Costs from Cancer Studies -- 5.1"Types of Costs Typical of Cancer Trials -- 5.1.1"Categorization of Health Resource Use -- 5.1.2"Resource Use Monitoring -- 5.1.3"Baseline Characteristics and Health Resource Use -- 5.1.4"Costs Determined by a Study Protocol -- 5.2"Perspective of Analysis and Costs Collection -- 5.3"Collecting Health Resource Use Across the Treatment Pathway -- 5.3.1"Time Horizon -- 5.4"Costing Methods: Micro versus Macro Approach -- 5.4.1"Average versus Marginal and Incremental Cost -- 5.4.2"Inflation -- 5.4.3"Time Preference and Discounting -- 5.5"Charges |
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5.5.1"Cost-to-Charge Ratios -- 5.5.2"Other Non-Medical Costs (e.g. Societal Costs) -- 5.6"Distribution of Costs -- 5.6.1"Transforming Cost Data -- 5.7"Handling Censored and Missing Costs -- 5.7.1"Strategies for Avoiding Missing Resource Data -- 5.7.2"Strategies for Analyzing Cost Data When Data Are Missing or Censored -- 5.7.3"Imputation Methods -- 5.8"Handling Future Costs -- 5.9"Case Report Forms and Health Resource Use -- 5.10"Statistical Analyses of Costs -- 5.11"Summary -- 5.12"Exercises for Chapter 5 -- 6: Designing Cost-Effectiveness into Cancer Trials -- 6.1"Introduction and Reasons for Collecting Economic Data in a Clinical Trial -- 6.2"Clinical Trial Designs for Cancer Studies -- 6.2.1"Clinical Trial Designs -- 6.2.2"Interim Analyses and Data Monitoring Committees (DMC) -- 6.3"Planning a Health Economic Evaluation in a Clinical Trial -- 6.3.1"Important Considerations When Designing a Cancer Study for Economic Evaluation -- 6.3.2"Integrating Economic Evaluation in a Clinical Trial: Considerations -- 6.3.3"Endpoints and Outcomes -- 6.3.3.1"Timing of Measurements -- 6.3.3.2"Trial Design -- 6.3.3.3"CRF Design -- 6.3.3.4"Sample Size Methods for Cost-Effectiveness -- 6.3.3.5"Sample Size Formulae for Cost-Effectiveness: Examples -- 6.3.4"Treatment Pathways -- 6.3.5"Time of Generic/Competition Entry -- 6.3.6"Treatment Compliance -- 6.3.7"Identify Subgroups/Heterogeneity -- 6.3.8"Early ICER/INMB -- 6.3.9"Multicenter Trials -- 6.4"Case Study of Economic Evaluation of Cancer Trials -- 6.4.1 TA516 Cabozanitib + Vandetanib -- 6.5"Summary -- 6.6"Exercises for Chapter 6 -- 7: Models for Economic Evaluation of Cancer -- 7.1"Types of Health Economic Models -- 7.2"Decision Tree Models -- 7.2.1"Further Possible Improvements to the Decision Model -- 7.3"Markov Models -- 7.4"Continuous Time Markov Models -- 7.5"The Partitioned Survival Model |
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7.5.1"Developing an Economic Model Using Patient-Level Data Using a Partitioned Survival Model Approach -- 7.5.1.1"Modeling the efficacy data (survival data) -- 7.5.2"Case Study of an Economic Model Using Patient-Level Data: A Partitioned Survival Model -- 7.5.3"Crossover -- 7.6"Summary of Cost-Effectiveness Models for Cancer Used in HTA Submissions -- 7.7"Summary -- 7.8"Exercises for Chapter 7 -- 8: Real-World Data in Cost-Effectiveness Studies on Cancer -- 8.1"Introduction to Real-World Data -- 8.2"Using RWD to Support Cost-Effectiveness Analysis -- 8.3"Strengths and Limitations of Using RWD to Support Cost-Effectiveness Analysis -- 8.3.1"Limitations -- 8.3.2"Internal Validity versus Generalizability -- 8.4"Sources for RWD Generation -- 8.4.1"Registries -- 8.4.2"Audits -- 8.4.3"Primary Care Databases: CPRD, THIN, QResearch -- 8.4.4"Insurance Claims Databases -- 8.4.5"Digital Data Sources, Social Media and Applications -- 8.4.6"Commercial Data Sources -- 8.4.7"Pragmatic Clinical Trials -- 8.4.8"Prospective Observational Research Studies -- 8.4.9"Case Control Studies -- 8.5"Using Cancer Registries -- 8.5.1"Examples of Registries in the UK for RWE -- 8.6"Statistical Analyses of RWD: Addressing Selection Bias -- 8.6.1"Propensity Score Modeling -- 8.6.2"Instrumental Variable Methods -- Results -- 8.7"Summary and Conclusion -- 8.8"Exercises for Chapter 8 -- 9: Reporting and Interpreting Results of Cost-Effectiveness Analyses from Cancer Trials -- 9.1"Interpreting Incremental Costs and QALYs -- 9.1.1"Informative Censoring -- 9.2"Interpreting Incremental QALYs -- 9.3"Relationship between Costs and QALYs -- 9.4"Interpreting the ICER and the Cost-Effectiveness Plane -- 9.4.1"Uncertainty -- 9.5"Presenting and Interpreting Results from Uncertainty Analysis -- 9.6"Bayesian Sensitivity Analysis -- 9.6.1"Limitations of the ICER and Using the INMB |
| Notes |
9.7"Presenting and Interpreting Results from Value of Information Analyses |
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Publisher supplied metadata and other sources |
| Subject |
Antineoplastic agents -- Development -- Economic aspects
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Clinical trials -- Economic aspects
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Medical economics.
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Economics, Medical
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Medical economics.
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| Form |
Electronic book
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| Author |
Crott, Ralph
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Bashir, Zahid
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| ISBN |
9781498761314 |
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1498761313 |
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9780429531842 |
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0429531842 |
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9780429546549 |
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0429546548 |
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9780429155826 |
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0429155824 |
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