ABSTRACT
Assessing benefits and risks is at the heart of decisions payers must make regarding access to medications authorized for marketing via the regulatory process. This common foundational use of benefit and risk by payers and regulators in their decision processes suggests an opportunity to harmonize the production and use of evidence regarding benefit and risk, and some evidence of harmonization is already beginning to emerge. However, unlike regulators, payers must also consider the cost of new treatments in their decisional framework, and this difference has sometimes stood in the way of mutual understanding and cooperation between regulators and payers. To better understand the role of benefit and risk in the decisions faced by
CONTENTS
10.1 Introduction ................................................................................................ 165 10.1.1 How Does the Payer Perspective Differ from the
Regulatory Perspective? ................................................................ 166 10.1.2 The Rise of Real-World Evidence in Payer Decision
Making ............................................................................................ 167 10.2 Role of Health Technology Assessment in Payer Decision
Making ........................................................................................................ 167 10.2.1 What Are the Components of an HTA? ...................................... 168 10.2.2 Tools and Conduct of HTA-How Are Benefit and Risk
Extrapolated to the Real World? .................................................. 169 10.2.3 Geographic Variability in the Application of HTA to Payer
Decision Making ............................................................................ 170 10.3 Recent Trends and Future Opportunities .............................................. 174
10.3.1 Trends in Payer Use of Evidence and HTA ................................ 174 10.3.2 Future Opportunities .................................................................... 175
10.4 Conclusions ................................................................................................. 177 References ............................................................................................................. 178
payers, we will consider their perspective and operating environment, and the role of health technology assessment (HTA) in their decision making. We will then outline recent trends in payers’ use of evidence and HTA, and highlight future opportunities for cooperation between payers and regulators.
