Presentation (CTI)

OBJECTIVES: This study aimed to develop a set of treatment attributes and corresponding levels for inclusion in a discrete choice experiment-based survey. The survey is intended to quantify preferences across Europe regarding treatment- and disease-related characteristics relevant to cystic fibrosis.
METHODS: A multi-stage, patient-centered approach was used, with input from an advisory board. First, semi-structured exploratory interviews were conducted with Flemish and Dutch adults with cystic fibrosis, children with cystic fibrosis, and caregivers to explore preferences regarding gene therapy. Second, scoring and ranking exercises were conducted with the same participant groups to identify patient-relevant treatment characteristics. Findings were complemented by a systematic review of preference studies. Third, an advisory board meeting with cystic fibrosis clinicians and gene therapy experts was held to select and refine the final attributes. Finally, a clinical trial database analysis was used to define the attribute levels.
RESULTS: Five attributes covering expected benefits and uncertainty were selected for inclusion in the survey: treatment frequency and duration of benefit, quality of life related to respiratory symptoms, quality of life related to digestive symptoms, uncertainty regarding the long-term risk of cancer, and uncertainty regarding the predictive role of organoids.
CONCLUSIONS: This study describes a patient-centered and evidence-informed process for developing treatment attributes for a preference survey in cystic fibrosis. The final set of attributes captures key dimensions of benefit and uncertainty relevant to future gene therapy decision-making. Next steps include survey validation through think-aloud interviews, followed by translation and dissemination through hospitals and patient organizations.