Australian IRD Community Shows High Interest, Low Knowledge in Gene Therapy: A Call for Education

Australian IRD Community Shows High Interest, Low Knowledge in Gene Therapy: A Call for Education

For individuals and families navigating the challenges of inherited retinal diseases (IRDs), the promise of gene therapy offers a beacon of hope. This emerging field holds the potential to halt or even reverse vision loss, a significant shift from previous prognoses. Understanding the community's perspectives on these groundbreaking treatments is crucial for effective advocacy, policy development, and patient education. A recent survey conducted in Australia sheds light on this very topic, revealing both enthusiastic interest and a critical need for enhanced understanding within the IRD community.

Published in Nature on October 2, 2022, the study, titled "Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia," provides the first comprehensive analysis of Australian IRD patients' views on gene therapy. The research aimed to assess patients' understanding of and interest in both currently approved and future hypothetical gene therapies for retinal dystrophy.

Key Findings from the Australian Survey:

• High Interest: A significant majority of survey respondents, 91.6% (or 92% according to some summaries), indicated they would likely accept gene therapy if it were available to them or their family members for IRD.
• Knowledge Gap: Despite high interest, only 28.3% of respondents felt they had good knowledge of gene therapy. Knowledge gaps were specifically identified regarding the methods and outcomes of gene therapy.
• Information Sources: Nearly half of the respondents (49.3%) obtained information about gene therapy from the internet, rather than from an ophthalmologist.
• Perceived Barriers: Almost 60% of respondents reported at least one barrier to their future uptake of the treatment, including out-of-pocket costs (30.5%) and fear of side effects (27%).
• Economic Value: Most respondents (79%) agreed that government subsidy for gene therapy would be an effective use of taxpayer money.
• Willingness to Travel: 77% of respondents indicated they would travel interstate for treatment.

This study, which included 681 participants with a mean age of 53.5 years, highlights a critical need for targeted education. While the enthusiasm for gene therapy is clear, the lack of comprehensive understanding could impact informed decision-making. Associate Professor Lauren Ayton noted that