OBJECTIVES: Stated preference studies, such as discrete choice experiments, are a robust way of collecting information about health preferences to inform drug development and healthcare decision-making. Such studies are traditionally a time and resource intensive process, often taking over 12 months to complete. The objective of this study was to demonstrate a pragmatic mixed-methods approach to systematically generate exploratory preference data for the treatment of a rare disease, generalized myasthenia gravis (gMG).

METHODS: Participants living with gMG (n=18) first received pre-read materials that included descriptions of six treatment attributes and a unique link to a brief online survey with a single risk threshold task. Following completion of the online survey, participants took part in qualitative focus groups to discuss their experience with gMG treatments and completed up to 3 threshold tasks, one of which repeated the threshold task from the online survey. This study took 4 months to complete.

RESULTS: The study elicited quantitative exploratory data on participants’ risk tolerance, as well as qualitative data on treatment expectations and other important treatment attributes and trade-offs participants were willing to make. Focus group discussions provided insights into participants’ choices in the threshold tasks, confirmed that all the attributes were relevant, and helped clarify what was important about the attributes.

CONCLUSIONS: Patient preference information is valuable for incorporating the patient perspective into the drug development process and medical decision-making. However, it is not always feasible to implement robust preference studies within the decision-making time frames. In such instances, such as internal decision-making or early clinical development decisions, where the same level of rigor in the data generation is not required, more pragmatic approaches, such as the mixed-methods approach applied here may provide sufficient preference information to fit the research needs of the study.