Economic Evaluation of Cancer Drugs: Using Clinical Trial and Real-World Data, 1st Edition (Hardback) book cover

Economic Evaluation of Cancer Drugs

Using Clinical Trial and Real-World Data, 1st Edition

By Iftekhar Khan, Ralph Crott, Zahid Bashir

Chapman and Hall/CRC

418 pages | 30 B/W Illus.

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Hardback: 9781498761307
pub: 2019-07-01
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Description

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.

Table of Contents

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

About the Authors

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.

About the Series

Chapman & Hall/CRC Biostatistics Series

Learn more…

Subject Categories

BISAC Subject Codes/Headings:
MAT029000
MATHEMATICS / Probability & Statistics / General
MED028000
MEDICAL / Epidemiology
MED071000
MEDICAL / Pharmacology