A multinational survey of potential participant perspectives on ocular gene therapy.
Alexis Ceecee ZhangMyra B McGuinessFred K ChenJohn R B GriggHeather G MackLauren N AytonPublished in: Gene therapy (2024)
Amidst rapid advancements in ocular gene therapy, understanding patient perspectives is crucial for shaping future treatment choices and research directions. This international cross-sectional survey evaluated knowledge, attitudes, and perceptions of ocular genetic therapies among potential recipients with inherited retinal diseases (IRDs). Survey instruments included 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. This study included 496 participant responses (89% adults with IRDs; 11% parents/guardians/carers) from 35 countries, with most from the United States of America (USA; 69%) and the United Kingdom (11%). Most participants (90%) indicated they would likely accept gene therapy if it was available, despite only 45% agreeing that they had good knowledge of gene therapy. The main sources of information were research registries (60% of participants) and the internet (61%). Compared to data from our recently published Australian national survey of people with IRDs (n = 694), USA respondents had higher knowledge of gene therapy outcomes, and Australian respondents indicated a higher perceived value of gene therapy treatments. Addressing knowledge gaps regarding outcomes and financial implications will be central to ensuring informed consent, promoting shared decision-making, and the eventual clinical adoption of genetic therapies.
Keyphrases
- gene therapy
- healthcare
- clinical trial
- mental health
- cross sectional
- primary care
- electronic health record
- optic nerve
- type diabetes
- systematic review
- physical activity
- depressive symptoms
- adipose tissue
- gene expression
- randomized controlled trial
- patient reported outcomes
- risk assessment
- social support
- climate change
- smoking cessation
- human health