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 and Marie Falahee
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Jorien Veldwijk: Erasmus University Rotterdam
Rachael Lynn DiSantostefano: Janssen Research & Development
Ellen Janssen: Janssen Research & Development
Gwenda Simons: University of Birmingham
Matthias Englbrecht: Freelance Healthcare Data Scientist
Karin Schölin Bywall: Uppsala University
Christine Radawski: Eli Lilly and Company
Karim Raza: University of Birmingham
Brett Hauber: Pfizer, Inc.
Marie Falahee: University of Birmingham

The Patient: Patient-Centered Outcomes Research, 2023, vol. 16, issue 6, No 6, 653 pages

Abstract: 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

Date: 2023
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DOI: 10.1007/s40271-023-00643-w

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