title: Patient-Focused Benefit-Risk Analysis to Inform Regulatory Decisions: The European Union Perspective.
abstract: Regulatory decisions are often based on multiple clinical end points, but the perspectives used to judge the relative importance of those end points are predominantly those of expert decision makers rather than of the patient. However, there is a growing awareness that active patient and public participation can improve decision making, increase acceptance of decisions, and improve adherence to treatments. The assessment of risk versus benefit requires not only information on clinical outcomes but also value judgments about which outcomes are important and whether the potential benefits outweigh the harms. There are a number of mechanisms for capturing the input of patients, and regulatory bodies within the European Union are participating in several initiatives. These can include patients directly participating in the regulatory decision-making process or using information derived from patients in empirical studies as part of the evidence considered. One promising method that is being explored is the elicitation of "patient preferences." Preferences, in this context, refer to the individual's evaluation of health outcomes and can be understood as statements regarding the relative desirability of a range of treatment options, treatment characteristics, and health states. Several methods for preference measurement have been proposed, and pilot studies have been undertaken to use patient preference information in regulatory decision making. This article describes how preferences are currently being considered in the benefit-risk assessment context, and shows how different methods of preference elicitation are used to support decision making within the European context.
authors.affiliation: Health Economics and Health Care Management, Hochschule Neubrandenburg, Neubrandenburg, Germany. Electronic address: muehlbacher@hs-nb.de.
authors.forename: Axel C
authors.initials: AC
authors.lastname: Mühlbacher
authors.affiliation: Health Economics and Health Care Management, Hochschule Neubrandenburg, Neubrandenburg, Germany.
authors.forename: Christin
authors.initials: C
authors.lastname: Juhnke
authors.affiliation: Department of Epidemiology, University of Groningen, The Netherlands.
authors.forename: Andrea R
authors.initials: AR
authors.lastname: Beyer
authors.affiliation: Science Policy and Research, National Institute for Health and Care Excellence, London, UK.
authors.forename: Sarah
authors.initials: S
authors.lastname: Garner
mesh_terms: D003657:Decision Making; D005062:European Union; D033161:Government Regulation; D057240:Patient Preference; D055553:Prescription Drugs; D018570:Risk Assessment / Q000379:methods*
chemical_list: D055553:Prescription Drugs
keywords: benefit-risk assessment; patient involvement; regulatory decision making