VALUE BASED PRICING IN PHARMACEUTICALS
Prof. Dr. F. Cankat TULUNAY
CONTENTS
Foreword
Chapter I. Historical and Conceptual Foundations of Value-Based Pricing
1.1 Why Are We Discussing This Topic So Intensively?
1.2 Historical Development of Drug Pricing
1.3 The Cost-Based Era
1.4 The Emergence of Pharmacoeconomics
1.5 The Emergence of Value-Based Pricing
1.6 A Paradigm Shift in Drug Pricing: From Cost-Based to Value-Based Pricing
1.7 The Sovaldi Event: The Beginning of the Modern VBP Era
1.8 The Era of Gene Therapies, Cellular Therapies, and Million-Dollar Drugs
Chapter II. QALY, ICER, Health Technology Assessment, and Outcomes Research
2.1 QALY: The Most Famous and Most Controversial Concept in Health Economics
2.2 What Is a QALY?
2.3 An Example of QALY Calculation
2.4 Why Is QALY Important?
2.5 How Are Utility Values Obtained?
2.6 Criticisms of QALY
2.7 What Can We Use Instead of QALY?
2.8 Beyond QALY
2.9 From QALY to ICER
2.10 ICER: The Most Powerful and Most Controversial Indicator in Health Economics
2.11 ICER and Threshold Values
2.12 Limitations of ICER
2.13 Ethical Debates Surrounding ICER
2.14 From ICER to Health Technology Assessment
2.15 Health Technology Assessment (HTA)
2.16 Main Components of HTA
2.17 NICE: The Global Symbol of HTA
2.18 ISPOR and Outcomes Research
2.19 Real-World Data (RWD) and Real-World Evidence (RWE)
2.20 Randomized Controlled Trials versus Real-World Evidence
2.21 From Efficacy to Effectiveness
2.22 The Central Question of Health Economics
Chapter III. Value-Based Pricing Models Around the World
3.1 Does Every Country Define Value in the Same Way?
3.2 United Kingdom and NICE
3.3 Managed Access Agreements
3.4 End-of-Life Treatments and the Rule of Rescue
3.5 Germany and IQWiG
3.6 Canada and CADTH
3.7 Australia and PBAC
3.8 France and HAS
3.9 Italy and Outcome-Based Payment Models
3.10 Risk Sharing, Cost Sharing, and Payment by Results
3.11 The United States
3.12 The ICER Institute and Value Debates in the United States
3.13 The Inflation Reduction Act
3.14 Medicare Drug Price Negotiation Program
3.15 The Most Favored Nation Approach
3.16 Value-Based Purchasing in the United States
Chapter IV. Risk-Sharing and Outcome-Based Reimbursement Models
4.1 Origins of Risk Sharing
4.2 Financial Risk-Sharing Models
4.3 Outcome-Based Risk-Sharing Models
4.4 Coverage with Evidence Development (CED)
4.5 Managed Entry Agreements
4.6 European Experience
4.7 Risk Sharing in Rare Diseases and Gene Therapies
4.8 The Future of Reimbursement Models
Chapter V. Ethical Dimensions and Criticisms of Value-Based Pricing
5.1 Can Human Life Be Assigned a Price?
5.2 QALY and Ethical Debates
5.3 Age Discrimination
5.4 Disability-Related Debates
5.5 Rare Diseases and the Problem of Justice
5.6 The Rule of Rescue
5.7 Evaluating End-of-Life Therapies
5.8 The Right to Health and Resource Allocation
Chapter VI. Value-Based Pricing or Value-Based Marketing?
6.1 The Misuse of the Concept of Value
6.2 Price Anchoring
6.3 Artificial Comparator Selection
6.4 Surrogate Endpoints
6.5 Model Manipulation
6.6 Publication Bias
6.7 Lobbying, Stakeholder Influence, and Corruption
6.8 Public Resources, Private Profits
6.9 Arguments of the Supporters of Value-Based Pricing
6.10 How Should True Value Be Measured?
Chapter VII. Orphan Drugs and Rare Diseases
7.1 The Orphan Drug Act
7.2 Economics of Orphan Drugs
7.3 Ultra-Rare Diseases
7.4 HTA and Rare Diseases
7.5 Gene Therapies and Orphan Drugs
7.6 Sustainability Challenges
Chapter VIII. Gene Therapies, Cellular Therapies, and the Future of Value
8.1 Luxturna and the First Gene Therapies
8.2 Zolgensma and the Two-Million-Dollar Drug
8.3 Lenmeldy and the World's Most Expensive Medicines
8.4 Publicly Funded Science and Private Profit
8.5 Million-Dollar Therapies and Global Inequality
8.6 CAR-T Therapies and a New Paradigm
8.7 Hemgenix and New Records
8.8 The Limitations of QALY in Gene Therapies
8.9 Outcome-Based Payment Models
8.10 Artificial Intelligence and Future HTA Systems
8.11 Personalized Medicine and a New Definition of Value
Chapter IX. Pharmacoeconomics and Value-Based Pricing in Turkey
9.1 Turkey's Introduction to Pharmacoeconomics
9.2 Where Are We Today in Turkey?
9.3 Can Turkey Become a Regional HTA Center?
9.4 Future Priorities for Turkey
Chapter X. The Future of Value-Based Pricing
10.1 The Most Favored Nation Approach and Global Consequences
10.2 Medicare Price Negotiations
10.3 Artificial Intelligence and Health Economics
10.4 Living HTA
10.5 Digital Biomarkers and a New Definition of Value
10.6 Personalized Medicine and Future Economic Models
10.7 What Comes After Gene Therapies?
10.8 The Future of Pharmacoeconomics
Author's Final Reflections
Pharmacoeconomics in Turkey
References