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

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  • Caroline M. Vass

    (The University of Manchester)

  • Katherine Payne

    (The University of Manchester)

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.

Suggested Citation

  • Caroline M. Vass & Katherine Payne, 2017. "Using Discrete Choice Experiments to Inform the Benefit-Risk Assessment of Medicines: Are We Ready Yet?," PharmacoEconomics, Springer, vol. 35(9), pages 859-866, September.
  • Handle: RePEc:spr:pharme:v:35:y:2017:i:9:d:10.1007_s40273-017-0518-0
    DOI: 10.1007/s40273-017-0518-0
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    1. Thesis Thursday: David Mott
      by Chris Sampson in The Academic Health Economists' Blog on 2019-04-18 06:00:33

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    2. Rotaris, Lucia & Giansoldati, Marco & Scorrano, Mariangela, 2021. "The slow uptake of electric cars in Italy and Slovenia. Evidence from a stated-preference survey and the role of knowledge and environmental awareness," Transportation Research Part A: Policy and Practice, Elsevier, vol. 144(C), pages 1-18.
    3. David J. Mott & Laura Ternent & Luke Vale, 2023. "Do preferences differ based on respondent experience of a health issue and its treatment? A case study using a public health intervention," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(3), pages 413-423, April.
    4. Vass, Caroline M. & Boeri, Marco & Poulos, Christine & Turner, Alex J., 2022. "Matching and weighting in stated preferences for health care," Journal of choice modelling, Elsevier, vol. 44(C).
    5. Bennett Levitan & A. Brett Hauber & Marina G. Damiano & Ross Jaffe & Stephanie Christopher, 2017. "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 Patient: Patient-Centered Outcomes Research, Springer;International Academy of Health Preference Research, vol. 10(5), pages 531-536, October.

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