Using Discrete Choice Experiments to Inform the Benefit-Risk Assessment of Medicines: Are We Ready Yet?

Abstract

There is emerging interest in the use of discrete choice experiments as a means of quantifying the perceived balance between benefits and risks (quantitative benefit-risk assessment) of new healthcare interventions, such as medicines, under assessment by regulatory agencies. For stated preference data on benefit-risk assessment to be used in regulatory decision making, the methods to generate these data must be valid, reliable and capable of producing meaningful estimates understood by decision makers. Some reporting guidelines exist for discrete choice experiments, and for related methods such as conjoint analysis. However, existing guidelines focus on reporting standards, are general in focus and do not consider the requirements for using discrete choice experiments specifically for quantifying benefit-risk assessments in the context of regulatory decision making. This opinion piece outlines the current state of play in using discrete choice experiments for benefit-risk assessment and proposes key areas needing to be addressed to demonstrate that discrete choice experiments are an appropriate and valid stated preference elicitation method in this context. Methodological research is required to establish: how robust the results of discrete choice experiments are to formats and methods of risk communication; how information in the discrete choice experiment can be presented effectually to respondents; whose preferences should be elicited; the correct underlying utility function and analytical model; the impact of heterogeneity in preferences; and the generalisability of the results. We believe these methodological issues should be addressed, alongside developing a ‘reference case’, before agencies can safely and confidently use discrete choice experiments for quantitative benefit-risk assessment in the context of regulatory decision making for new medicines and healthcare products.

Patients' preferences in periodontal disease treatment elicited alongside an IQWiG benefit assessment: a feasibility study

“…Vass et al discussed a variety of methodological questions for further DCE research, including, for example, the framing of risks or probabilities, the influence of personal characteristics on preferences, and how to deal with heterogeneous preferences, a specification of whose preferences should be surveyed. 46 Solving all these still unresolved issues is a prerequisite before regular inclusion of preference data into HTA. Vass et al in this respect call for the development of a “reference case” to be able to “safely and confidently use discrete choice experiments” in HTA.…”

“…Vass et al in this respect call for the development of a “reference case” to be able to “safely and confidently use discrete choice experiments” in HTA. 46 …”

A Picture is Worth a Thousand Words: The Role of Survey Training Materials in Stated-Preference Studies

“…Approval for the study was obtained from The University of Manchester's Research Ethics Committee. inform regulatory decisions [8,9]. However, for DCEs to be used in decision making, they must be robust and produce data that minimize bias from either their hypothetical nature or other sources [10].…”

The Ball is in Your Court: Agenda for Research to Advance the Science of Patient Preferences in the Regulatory Review of Medical Devices in the United States

“…The CDRH guidance on incorporating patient preference information in regulatory submissions identifies multiple factors that industry sponsors should consider when developing a patient preference study [6]. However, researchers have noted that there remain many key questions to answer before decision makers can be confident in using patient-preference information to inform regulatory benefit-risk assessments [25]. In addition, MDIC identified several areas in which additional work may be necessary to understand the properties of patient preference methods and the implications of these properties for the use of patient preference information for regulatory purposes [3].…”