Exploring perceptions of using preference elicitation methods to inform clinical trial design in rheumatology: A qualitative study and OMERACT collaboration.

BACKGROUND: Clinical trial design requires value judgements and understanding patient preferences may help inform these judgements, for example when prioritizing treatment candidates, designing complex interventions, selecting appropriate outcomes, determining clinically important thresholds, or weighting composite outcomes. Preference elicitation methods are quantitative approaches that can estimate patients' preferences to quantify the absolute or relative importance of outcomes or other attributes relevant to the decision context. We aimed to explore stakeholder perceptions of using preference elicitation methods to inform judgements when designing clinical trials in rheumatology. METHODS: We conducted 1-on-1 semi-structured interviews with patients with rheumatic diseases and rheumatology clinicians/researchers, recruited using purposive and snowball sampling. Participants were provided pre-interview materials, including a video and a document, to introduce the topic of preference elicitation methods and case examples of potential applications to clinical trials. Interviews were conducted via Zoom and were audio-recorded and transcribed. We used thematic analysis to analyze our data. RESULTS: We interviewed 17 patients and 9 clinicians/researchers, until data and inductive thematic saturation were achieved within each group. Themes were grouped into overall perceptions, barriers, and facilitators. Patients and clinicians/researchers generally agreed that preference elicitation studies can improve clinical trial design, but that many considerations are required around preference heterogeneity and feasibility. A key barrier identified was the additional resources and expertise required to measure and incorporate preferences effectively in trial design. Key facilitators included developing guidance on how to use preference elicitation to inform trial design, as well as the role of external decision-makers in developing such guidance, and the need to leverage the movement towards patient engagement in research to encourage including patient preferences when designing trials. CONCLUSION: Our findings allowed us to consider the potential applications of patient preferences in trial design according to stakeholders within rheumatology who are involved in the trial process. Future research should be conducted to develop comprehensive guidance on how to meaningfully include patient preferences when designing clinical trials in rheumatology. Doing so may have important downstream effects for shared decision-making, especially given the chronic nature of rheumatic diseases.

The Application of Preference Elicitation Methods in Clinical Trial Design to Quantify Trade-Offs: A Scoping Review.

BACKGROUND AND OBJECTIVE: Patients can express preferences for different treatment options in a healthcare context, and these can be measured with quantitative preference elicitation methods. OBJECTIVE: Our objective was to conduct a scoping review to determine how preference elicitation methods have been used in the design of clinical trials. METHODS: We conducted a scoping review to identify primary research studies, involving any health condition, that used quantitative preference elicitation methods, including direct utility-based approaches, and stated preference studies, to value health trade-offs in the context of clinical trial design. Studies were identified by screening existing systematic and scoping reviews and with a primary literature search in MEDLINE from 2010 to the present. We extracted study characteristics and the application of preference elicitation methods to clinical trial design according to the SPIRIT checklist from primary studies and summarized the findings descriptively. RESULTS: We identified 18 eligible studies. The included studies applied patient preferences to five areas of clinical trial design: intervention selection (n = 1), designing N-of-1 trials (n = 1), outcome selection and weighting composite and ordinal outcomes (n = 12), sample size calculations (n = 2), and recruitment (n = 2). Using preference elicitation methods led to different decisions being made, such as using preference-weighted composite outcomes instead of equally weighted composite outcomes. CONCLUSION: Preference elicitation methods are infrequently used to design clinical trials but may lead to changes throughout the trial that could affect the evidence generated. Future work should consider measurement challenges and explore stakeholder perceptions.