Presentation (CTI)

OBJECTIVES: To co-design a discrete choice experiment (DCE) that quantifies patients’ preferences for emerging gene therapy for retinal disease in Germany, Ireland and Spain.
METHODS: We first conducted a rapid literature review and online workshops with patients from three countries (two patients from Germany, three from Ireland and one from Spain) to inform and co-design the attributes and corresponding levels of the DCE survey. Patients complete choice tasks asking them to choose between different potential gene therapy profiles as well as an opt-out option. The trade-offs patients make regarding treatment attributes, as well as their willingness to pay (WTP) for gene therapy will be estimated using mixed logit models.
RESULTS: Through the rapid review and workshops, we identified a total number of five attributes. These included the effect gene therapy would have on vision, duration of treatment effect, risk of side effects, chance of responding to the gene therapy, as well as out-of-pocket costs. Preliminary results suggest that there was a need to define the effect of gene therapy in terms of visual function, encompassing the ability to read information, navigate one’s surroundings and see in the distance. In addition, patients often perceived potential long-term risks associated with gene therapy as more uncertain than short-term risks.
CONCLUSIONS: Given the expected future market entry of several retinal gene therapies, patients’ treatment preferences will have a considerable impact on how often financially constrained health systems deliver these costly therapies. This study adds further evidence by identifying attributes of retinal gene therapies and the associated value of these therapies. This will inform decision-makers as to the adequate delivery of such resource-intensive technologies to patients.