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.

Patient preferences on rheumatoid arthritis second-line treatment: A discrete choice experiment of Swedish patients

“…Clinicians need to understand their patients' preferences and perspectives when informing their patients about potential RA treatments so their patients can make decisions about their treatment that are aligned with their preferences [9,13,14]. Quantitative assessments of patient preferences have the potential to support both clinicians and regulators when they consider patient perspective [7,11].…”

Utilising Benefit-Risk Assessments within Clinical Trials – A Protocol for the BRAINS project

“…This is therefore providing a comparison of competing treatments over multiple outcomes. B-R methodology is already commonly used within the regulatory setting where it is important for regulators to be able to evaluate the benefits of a drug against its harms (4). In 2007, the European Medicine Agency (EMA) published a report which showed the potential value of existing B-R models and methods (5,6) and further created decision-making models for use in this area (7).…”

Attribute Non-Attendance in Discrete-Choice-Experimenten

“…Die Berücksichtigung von Patientenpräferenzen kann Entscheidungen über die Inanspruchnahme von Gesundheitsleistungen verbessern, die Patientenorientierung stärken und langfristig zur Verbesserung von Behandlungsergebnissen führen [5]. Auch im nationalen und internationalen Kontext hat die Berücksichtigung von Patientenpräferenzen in den vergangenen Jahren enorm an Bedeutung gewonnen [6,7]. Auf regulatorischer Ebene der Entscheidungsfindung ist die Europäische Arzneimittelbehörde (EMA) in einer Pilotstudie zu dem Ergebnis gekommen, dass Methoden der Präferenzmessung geeignet sind, um unterschiedliche Stakeholder an Entscheidungsprozessen zu beteiligen [6].…”