Cancer is a major healthcare burden across the world and impacts not only the people diagnosed with various cancers but also their families, carers, and healthcare systems. With advances in the diagnosis and treatment, more people are diagnosed early and receive treatments for a disease where few treatments options were previously available. As a result, the survival of patients with cancer has steadily improved and, in most cases, patients who are not cured may receive multiple lines of treatment, often with financial consequences for the patients, insurers and healthcare systems. Although many books exist that address economic evaluation, Economic Evaluation of Cancer Drugs using Clinical Trial and Real World Data is the first unified text that specifically addresses the economic evaluation of cancer drugs.
The authors discuss how to perform cost-effectiveness analyses while emphasising the strategic importance of designing cost-effectiveness into cancer trials and building robust economic evaluation models that have a higher chance of reimbursement if truly cost-effective. They cover the use of real-world data using cancer registries and discuss how such data can support or complement clinical trials with limited follow up. Lessons learned from failed reimbursement attempts, factors predictive of successful reimbursement and the different payer requirements across major countries including US, Australia, Canada, UK, Germany, France and Italy are also discussed. The book includes many detailed practical examples, case studies and thought-provoking exercises for use in classroom and seminar discussions.
Iftekhar Khan is a medical statistician and health economist and a lead statistician at Oxford Unviersity's Center for Statistics in Medicine. Professor Khan is also a Senior Research Fellow in Health Economics at University of Warwick and is a Senior Statistical Assessor within the Licensing Division of the UK Medicine and Health Regulation Agency.
Ralph Crott is a former professor in Pharmacoeconomics at the University of Montreal in Quebec, Canada and former head of the EORTC Health Economics Unit and former senior health economist at the Belgian HTA organization.
Zahid Bashir has over twelve years experience working in the pharmaceutical industry in medical affairs and oncology drug development where he is involved in the design and execution of oncology clinical trials and development of reimbursement dossiers for HTA submission.
1. Introduction to Cancer Cancer Epidemiology of Cancer Prognostic factors associated with cancer outcomes Economic Burden of Cancer Treatments for Cancer Important Economic Concepts for Cost-Effectiveness of Cancer interventions Health Economic Evaluation and Cancer Drug Development in Practice Efficacy versus Effectiveness Real World Data Economic versus Clinical Hypotheses Exercises 2. Introduction Important Common, Surrogate and Novel Cancer Endpoints Overall Survival Surrogate Endpoints HTAs with Surrogate Endpoints Emerging Tumour-Centred Endpoints Demonstrating Value from other cancer endpoints Exercises 3. Health Related Quality of Life for Cost-effectiveness Health Related Quality of Life (HRQoL) in cancer patients Measuring HRQoL for Economic Evaluation Constructing Utilities Quality Adjusted Life Years (QALYs) Economic Evaluation in the Absence of Utility Data: Mapping and Utility Studies Sensitivity and Responsiveness of EQ-D versus QLQ-C HRQoL for detecting improvement in cancer patients Measuring Post-Progression Utility: Some Approaches Plausible Post-Progression Utility Behaviour Non-Linear Models HRQoL issues in Health Technology Appraisals of Cancer Drugs Exercises 4. Introductory Statistical Methods for Economic Evaluation in Cancer Introduction Uncertainty and variability Distributions: Cost, Utility and Survival Data Important measures used in cancer trials Simulation: Bootstrapping and Monte-Carlo Simulation Analysing Data from cancer Trials Semi-Parametric Methods - The Cox PH model Parametric Methods: Modelling Survival Data for Extrapolation Advanced Modelling Techniques for Survival Data Issues in fitting models Handling Crossover, Treatment Switching and Subsequent anti-cancer therapy Data Synthesis and Network Meta Analyses Mixed Treatment Comparisons Assumptions for carrying out MTCs Exercises 5. Collecting and Analysis of Costs from Cancer Studies Types of costs typical of cancer trials Perspective of analysis and costs collection Collecting Health Resource use across the treatment pathway Costing methods: micro versus macro approach Charges Distribution of Costs Handling Censored and Missing Costs Strategies for avoiding missing resource data Strategies for analysing cost data when data are missing or censored Handling Future Costs Case Report Forms and Health Resource Use Statistical Analyses of Costs Exercises 6. Designing Cost-effectiveness into Cancer Trials Introduction and Reasons for Collecting Economic Data in a Clinical Trial Clinical Trial Designs for Cancer Studies Planning a Health Economic Evaluation in a Clinical Trial Important considerations when designing a cancer study for economic evaluation Integrating Economic Evaluation in a Clinical Trial: Considerations Figure : Clustered data within each centre Case study of Economic Evaluation of cancer trials Exercises 7. Models for Economic Evaluation of Cancer Types of Health Economic Models Decision Tree Models Markov Models Continuous time Markov Models The partitioned survival model Developing an economic model using patient level data using a partitioned survival model approach Case Study of an economic model using patient level data: a partitioned survival model Summary of Cost-Effectiveness Models for Cancer used in HTA submissions Exercises 8. Real World Data in Cost-Effectiveness studies on Cancer Introduction to Real World Data Using RWD to Support Cost-Effectiveness Analysis Strengths and Limitations of Using RWD to Support Cost-Effectiveness Analysis Internal Validity versus Generalizability Sources for RWD generation Using Cancer Registries Statistical Analyses of RWD: Addressing Selection Bias Propensity Score Modelling Instrumental Variable Methods Summary and Conclusion Exercises for Chapter 9. Reporting and Interpreting Results of Cost-effectiveness Analyses from Cancer trials Interpreting Incremental Costs and Utilities Interpreting Incremental QALYs Relationship between costs and QALYs Interpreting the ICER and the Cost-effectiveness Plane Presenting and interpreting results from uncertainty analyses Limitations of the ICER and using the INMB Presenting and interpreting results from Value of Information Analyses (VOI) Exercises 10. Important Lessons from Failed Reimbursement Attempts A List of rejected cancer drugs by NICE When it's useded Summary of criticisms of Economic Models of Cancer Factors Predictive of successful HTAs in Cancer The changing pace of the reimbursement environment Reimbursement and payer evidence requirements across different countries Pricing and reimbursement environment in United States Value based pricing (VBP) for Cancer Drugs Risk Sharing Scheme The Future of Cost-effectiveness of Cancer Treatments Future Research: Methodology Future Reimbursement Landscape
Iftekhar Khan is a medical statistician and health economist and a lead statistician at Oxford Unviersity's Center for Statistics in Medicine. Professor Khan is also a Senior Research Fellow in Health Economics at University of Warwick and is a Senior Statistical Assessor within the Licensing Division of the UK Medicine and Health Regulation Agency. Ralph Crott is a former professor in Pharmacoeconomics at the University of Montreal in Quebec, Canada and former head of the EORTC Health Economics Unit and former senior health economist at the Belgian HTA organization. Zahid Bashir has over twelve years experience working in the pharmaceutical industry in medical affairs and oncology drug development where he is involved in the design and execution of oncology clinical trials and development of reimbursement dossiers for HTA submission.
Reviews for Economic Evaluation of Cancer Drugs: Using Clinical Trial and Real-World Data
This book is highly recommended for readers searching for an introductory text to the world of health economic analysis. The authors provide timely examples from both clinical trials in oncology and subsequent real-world application, discussing implications of findings and how they could potentially be applied to make future trials and real-world applications more efficient. ~Nuru Noor, ISCB News