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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. "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

Economic Evaluation of Cancer Drugs: Using Clinical Trial and Real-World Data

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