Presentation (CTI)

OBJECTIVES: The primary objective of the study was to explore preferences regarding gene therapy among adults with CF, children with CF and their caregivers. The secondary objective was to identify decision-making drivers when choosing between standard of care and a hypothetical gene therapy.
METHODS: Semi-structured interviews were conducted to obtain in-depth insights into willingness to take gene therapy, participate in gene therapy trials, and decision-making drivers.
RESULTS: Views toward gene therapy differed among participants. Gene therapy-supportive patients emphasized the added value of a one-time, targeted treatment with the potential to improve respiratory symptoms and quality of life. Gene therapy-averse patients emphasized concerns about off-target effects, treatment burden, and the need for additional safety and efficacy evidence. Participants expressed their interest in participating in clinical trials, but this was conditional on the amount of available evidence and their medical need. In addition, the one-time nature and potential efficacy of gene therapy treatment were key decision-making drivers. However, participants also described how their health status, including disease severity and age, influenced the trade-offs they are willing to make between benefits and risks.
CONCLUSIONS: This interview study shows that gene therapy is seen positively among adults with cystic fibrosis, children with cystic fibrosis, and their caregivers, particularly due to its potential long-term effect. However, participants also expressed important concerns regarding uncertainty, off-target effects. The findings suggest that people with more severe disease may be more willing to accept gene therapy-related risks, which could inform future clinical trial design, patient selection, and early regulatory and reimbursement discussions.