ABSTRACT
In 2006, a preference study of patients’ risk tolerance was submitted to the US Food and Drug Administration (FDA) Tysabri Advisory Committee as part of the drug’s reapproval application. This submission marked the first time such evidence was included in support of drug licensing. The Center for Drug Evaluation and Research (CDER) currently is developing a benefit–risk evaluation framework to better communicate which benefits and risks are considered, how the available evidence is interpreted, and how the benefits and risks are weighed. FDA’s Center for Devices and Radiological Health (CDRH) issued guidance in 2012 and 2015 for conducting benefit–risk assessments which explicitly includes consideration of patients’ tolerance for risks and perspectives on benefits. A decision tool using patient-preference weights is being used to evaluate benefits and risks of new devices. This chapter provides a conceptual framework for quantifying patient benefit–risk tradeoff preferences, outlines procedures for eliciting valid preference weights for therapeutic benefits and harms, and reviews applications of these methods in two recent regulatory case studies for Duchenne’s muscular dystrophy and weight-loss devices. It concludes with an evaluation of prospects for integrating quantitative evidence 86on patients’ willingness to accept therapeutic risks in return for improved efficacy in regulatory decision making.
