Reflections on emerging therapies for inherited retinal diseases following a community education event: A qualitative exploration.

Abstract

To explore the inherited retinal diseases (IRD) communities' attitudes and perspective toward emerging therapies-specifically gene therapy, cell therapy, and retinal prostheses-following a community education event. At an in-person half-day IRD community education event, subject-matter experts presented the scientific foundations and current research developments related to these therapies. Following presentations, we invited attendees with lived experience to participate in an audio-recorded focus group to share their views and reflect on information presented. We conducted a thematic analysis of the focus group transcriptions to identify key themes. Our study was informed by a relativist ontology and constructivist epistemology. We conducted three focus groups, totaling 10 people with IRD and seven caregivers. Our analysis yielded four themes: (1) The drive for therapy, describing the burden of IRD and desire for any therapy; (2) navigating hope and uncertainty, highlighting the emotional complexity of disease progression and need to balance hope with realistic expectations; (3) personalized communication, emphasizing the value of tailored communication from trusted health professionals; and (4) transparency in information sharing, underscoring the importance of ongoing research updates. Our participants shared a strong desire for effective treatment, regardless of the therapeutic approach. Limited options and anticipated timeline to develop gene-specific therapies led many to view gene-agnostic approaches as more viable within their lifetime. Ongoing research updates were deemed valuable for fostering acceptance of emerging therapies and supporting treatment decision-making.