Debating Modern Medical Technologies : The Politics of Safety, Effectiveness, and Patient Access

"This book analyzes policy fights about what counts as good evidence of safety and effectiveness when it comes to new health care technologies in the United States and what political decisions mean for patients and doctors. Medical technologies often promise to extend and improve quality of life but come with many questions: Are they safe and effective? Are they worth the cost? When should they be allowed on the market, and when should Medicare, Medicaid, and private insurance companies be required to pay for drugs, devices, and diagnostic tests? Using case studies of disputes about the value of mammography screening; genetic testing for disease risk; brain imaging technologies to detect biomarkers associated with Alzheimer's disease; cell-based therapies; and new, expensive drugs, Maschke and Gusmano illustrate how scientific disagreements about what counts as good evidence of safety and effectiveness are often swept up in partisan fights over health care reform and battles among insurance and health care companies, physicians, and patient advocates. Debating Modern Medical Technologies: The Politics of Safety, Effectiveness, and Patient Access reveals stakeholders' differing values and interests regarding patient choice, physician autonomy, risk assessment, government intervention in medicine and technology assessment, and scientific innovation as a driver of national and global economies. It will help readers to understand the nature and complexity of past and current policy disagreements and their effects on patients. Features: Helps readers to understand the political, economic, and ideological dimensions of disputes about health technology assessment and the implications of various policy approaches for patients and the health care system; Includes discussion of disputes related to hot topics such as stem cell therapies, mammography screening, genomic tests, breast cancer drugs, and Hepatitis C medications; Discusses interest group pressure on government from large pharmaceutical companies and medical device manufacturers; Connects to current political rhetoric about speeding up the availability of technology" -- From the publisher Cover 1 Title 4 Copyright 5 Contents 6 Acknowledgments 8 Chapter 1: Evidence, Politics, and Medical Care Technology 10 Chapter 2: Evolving Public- and Private-Payer Approaches to Evidence and Health Technology Assessment 22 Evidence and the Influence of Scientific Medicine 23 From Cost Crisis to Value for Money 25 Expanding the Federal Government’s Role in Health Technology Assessment 28 Political Attacks on Health Services Research 30 Renewed Enthusiasm for Comparative Effectiveness Research 32 Medicare’s Renewed Interest in Evidence of Effectiveness 35 Evidence of Value for Private Payers 37 Conclusion 38 Chapter 3: Mammography Screening: Vested Interests and Polarization 39 Breast Cancer Trends in the United States 40 Breast Cancer Advocacy and the Role of Mammography Screening 41 Mammography Screening 44 Concerns About Underuse and Overuse of Screening 45 The 2009 Mammography Screening Recommendations 49 The Problem with Disinvestment 51 Mammography and the Limited Focus of Breast Cancer Advocacy 52 The Limits and Politics of Weighting Risks 53 Obamacare, Rationing, and Hyperpartisanship 54 Conclusion 55 Chapter 4: When Medicare Said No for Amyloid PET Imaging 57 Imaging the Brain for Amyloid Plaque 58 Asking for CMS Coverage 59 CMS Decision-Making and Coverage Criteria 60 Coverage Criteria and the NCD Approach 60 The CED Pathway 61 The NCD for Amyloid PET Imaging 62 Advocacy Positions Regarding Amyloid PET Coverage 63 The NCD Outcome 65 Responses to the Final Decision 67 The IDEAS Study 68 Conclusion 69 Chapter 5: Placing a Value on Cure: Lessons from the New Generation of Hepatitis C Drugs 72 Incidence and Consequences of Hepatitis C 73 What Is Hepatitis C? 73 How Many People Are Infected with Hepatitis C? 74 What Are the Characteristics of People Who Are Infected? 74 Evolution of Hepatitis C Treatment 75 Public- and Private-Payer Coverage Policies 77 Implications for Health Care Spending 78 The Advocacy Campaign for Expanded Access 79 A Response to Stigmatized Patients? 81 Role of the Courts 81 Price Negotiation and Competition 82 Lessons from the New Generation of Hepatitis C Drugs 82 Drug Company Responses to Price Complaints 83 How to Evaluate Value in the Context of Cure? 85 Loss of Innovation 86 Conclusion 87 Chapter 6: Avastin and the Politics of Accelerated Approval 89 The Challenge of Treating Advanced Breast Cancer 91 Drug Regulation in the United States: From Caveat Emptor to Accelerated Approval 92 Progressive Era Reforms 92 The Food, Drug, and Cosmetic Act of 1938 93 The 1962 Kefauver-Harris Drug Amendments 94 Expedited Approval and Expanded Access 96 Conditional Approval to Use Avastin for Treating Advanced Breast Cancer 98 Reversing Course 99 June 2011 FDA Hearing 100 Conclusions: Avastin and the Push for Faster Approval 106 Chapter 7: The Path to the Clinic for Stem Cell and Other Regenerative Medicine Interventions 108 FDA Regulation of Cellular Products 110 The 2001 Rule 110 FDA’s Initial Enforcement Actions 111 FDA Clarifies the Regulatory Approach 113 Stem Cells and Right-to-Try Laws 115 Right-to-Try in the States 116 Right-to-Try in Congress 117 Regenerative Medicine and the 21st Century Cures Act 119 Conflicting Interest Group Positions 120 From REGROW to the Cures Act 121 The New Political and Regulatory Landscape 121 Gottlieb’s First Steps 122 FDA’s New Policy Framework 123 Conclusion 125 Chapter 8: Conclusion: The 21st Century Cures Act and the Future of Health Technology Assessment 127 The Importance of Framing in Scientific Disputes 130 Who Should Have the Power to Decide? 131 Complex Coalitions and Competition among Powerful Interest Groups 132 Paying for Technology and Disagreements about Public Priorities 132 Final Reflections 136 Public Engagement 137 Access to Postapproval Data 140 Public and Private Payer Transparency 141 Looking Forward 141 Notes 144 Bibliography 186 Index 220 About the Authors 226 This book analyzes policy fights about what counts as good evidence of safety and effectiveness for new health care technologies in the U.S. and how political decisions affect patients and doctors. It discusses stakeholders' differing values and interests regarding patient choice, physician autonomy, risk assessment, government intervention in medicine and technology assessment, and scientific innovation as a driver of national and global economies