Attitudes to Gene Therapy for the Eye – A Global Survey

Abstract

Purpose : With one regulatory-approved gene therapy for RPE65-associated retinal degeneration, and several others in the pipeline, it is important to understand potential recipients’ perspectives on these treatments, interest in uptake, and barriers to access. This survey aimed to capture current attitudes to ocular gene therapy in a global cohort of people with inherited retinal disease (IRD).

Methods : An online survey was distributed to adults with an IRD and parents/carers of those affected. The survey included demographic data and the Attitudes to Gene Therapy-Eye (AGT-Eye), EQ-5D-5L, National Eye Institute Visual Functioning Questionnaire (NEI-VFQ-25), and Patient Attitudes to Clinical Trials (PACT-22) instruments. Data was then compared to previously published Australian data from our group (n=694).

Results : Between 13 April and 25 July 2023, 496 participants completed the survey (89% adults with IRDs, mean age 55.6 [standard deviation: 14.4] years, 11% parents/guardians/carers, mean age 45.3 [11.0] years). Gender was equal between respondents overall (52% female), but more female parents/carers participated than male parents (69%). Respondents were from 35 countries, with most from the United States of America (USA; 69%) and United Kingdom (11%). Most participants (90%) indicated they would likely accept gene therapy if it was available to them or family members, but less than half (45%) felt that they had a good understanding of the technology. The main sources of information were research registries (60% participants) and the internet (61%). When compared to previous Australian data, we found that the USA respondents had a higher self-rated knowledge of ocular gene therapy, but reported lower perceived value of therapy scores.

Conclusions : This expansion to a previously published study in Australians with IRDs has provided further evidence that people with IRDs are highly motivated to access gene therapy. In the global cohort, representing over 35 countries, most respondents had highly positive views about gene therapy, and identified limited barriers for uptake. Further education is required for patients with IRDs in order to prepare them for the emerging treatments.

This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.