Can the General Public Be a Proxy for an “At-Risk” Group in a Patient Preference Study? A Disease Prevention Example in Rheumatoid Arthritis

Rachel L DiSantostefano*, G. Simons, M. Englbrecht, Jennifer H. Humphreys, Ian N. Bruce, K. Schölin Bywall, C. Radawski, K. Raza, M. Falahee, J. Veldwijk

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

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Abstract

Background: When selecting samples for patient preference studies, it may be difficult or impractical to recruit participants who are eligible for a particular treatment decision. However, a general public sample may not be an appropriate proxy.

Objective: This study compares preferences for rheumatoid arthritis (RA) preventive treatments between members of the general public and first-degree relatives (FDRs) of confirmed RA patients to assess whether a sample of the general public can be used as a proxy for FDRs.

Methods: Participants were asked to imagine they were experiencing arthralgia and had screening tests indicating a 60% chance of developing RA within 2 yrs. Using a discrete choice experiment, participants were offered a series of choices between no treatment and 2 unlabeled hypothetical treatments to reduce the risk of RA. To assess data quality, time to complete survey sections and comprehension questions were assessed. A random parameter logit model was used to obtain attribute-level estimates, which were used to calculate relative importance, maximum acceptable risk (MAR), and market shares of hypothetical preventive treatments.

Results: The FDR sample (n = 298) spent more time completing the survey and performed better on comprehension questions compared with the general public sample (n = 982). The relative importance ranking was similar between the general public and FDR participant samples; however, other relative preference measures involving weights including MARs and market share differed between groups, with FDRs having numerically higher MARs.

Conclusion: In the context of RA prevention, the general public (average risk) may be a reasonable proxy for a more at-risk sample (FDRs) for overall relative importance ranking but not weights. The rationale for a proxy sample should be clearly justified.
Original languageEnglish
Pages (from-to)189-202
Number of pages14
JournalMedical Decision Making
Volume44
Issue number2
Early online date19 Jan 2024
DOIs
Publication statusPublished - Feb 2024

Bibliographical note

Financial Support:
The authors disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: Financial support for this study was provided in part by a grant the Innovative Medicines Initiative (IMI) 2 Joint Undertaking under grant agreement No. 115966. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation program and European Federation of Pharmaceutical Industries and Associations (EFPIA). K. Raza is supported by the National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre. The funding agreement ensured the authors’ independence in designing the study, interpreting the data, writing, and publishing the report.

Keywords

  • stated preferences
  • proxy respondents
  • recruitment
  • sampling
  • patient preferences
  • rheumatoid arthritis

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