Why Should Regulators Consider Using Patient Preferences in Benefit-risk Assessment?

There is an increased interest in including the patient perspective in regulatory decision making. At present, regulatory agencies such as the European Medicines Agency (EMA) in the EU and the US FDA use a combination of patient consultation and patient participation in decision committees to ensure that the patient perspective is taken into account in the decision-making process. A relatively new area of interest is the assessment of quantitative patient preferences in larger patient populations. The aim of preference elicitation is to estimate the impact and importance of known adverse effects of a new drug compared with its benefits from a patient perspective, a trade-off that is now implicitly made by the decision committee. The opportunity offered by stated preference methods is to elicit preferences in a large and representative sample of the patient population, thereby improving the reliability and validity of the preferences itself. This paper discusses the potential of patient-based preference assessment of benefits and risks in the approval process for new healthcare technologies.