Maximum Acceptable Risk Estimation Based on a Discrete Choice Experiment and a Probabilistic Threshold Technique

Jorien Veldwijk*, Rachael Lynn DiSantostefano, Ellen Janssen, Gwenda Simons, Matthias Englbrecht, Karin Schölin Bywall, Christine Radawski, Karim Raza, Brett Hauber, Marie Falahee

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Abstract

Objective: We aimed to empirically compare maximum acceptable risk results estimated using both a discrete choice experiment (DCE) and a probabilistic threshold technique (PTT). Methods: Members of the UK general public (n = 982) completed an online survey including a DCE and a PTT (in random order) measuring their preferences for preventative treatment for rheumatoid arthritis. For the DCE, a Bayesian D-efficient design consisting of four blocks of 15 choice tasks was constructed including six attributes with varying levels. The PTT used identical risk and benefit attributes. For the DCE, a panel mixed-logit model was conducted, both mean and individual estimates were used to calculate maximum acceptable risk. For the PTT, interval regression was used to calculate maximum acceptable risk. Perceived complexity of the choice tasks and preference heterogeneity were investigated for both methods. Results: Maximum acceptable risk confidence intervals of both methods overlapped for serious infection and serious side effects but not for mild side effects (maximum acceptable risk was 32.7 percent-points lower in the PTT). Although, both DCE and PTT tasks overall were considered easy or very easy to understand and answer, significantly more respondents rated the DCE choice tasks as easier to understand compared with those who rated the PTT as easier (7-percentage point difference; p < 0.05). Conclusions: Maximum acceptable risk estimate confidence intervals based on a DCE and a PTT overlapped for two out of the three included risk attributes. More respondents rated the DCE as easier to understand. This may suggest that the DCE is better suited in studies estimating maximum acceptable risk for multiple risk attributes of differing severity, while the PTT may be better suited when measuring heterogeneity in maximum acceptable risk estimates or when investigating one or more serious adverse events.

Original languageEnglish
Pages (from-to)641-653
Number of pages13
JournalPatient
Volume16
Issue number6
Early online date30 Aug 2023
DOIs
Publication statusPublished - Nov 2023

Bibliographical note

Funding Information:
This study formed part of the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project. The PREFER project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No. 115966. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA. Karim Raza is supported by the National Institute for Health and Care Research Birmingham Biomedical Research Centre. The views expressed are those of the authors and not necessarily those of the National Institute for Health and Care Research or the Department of Health and Social Care. The funder had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.

Publisher Copyright: © 2023, The Author(s).

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