Using the nominal group technique to priortise the burden of geographic atrophy and treatment expectations of therapies: perspectives from patient leaders
Authors: Paudel Nabin , Moran Ellen Margaret , Stafford Sinead , McVicker Kelly , Stratieva Petia , Daly Avril 
Published: August 2024

Abstract

Background Age-related macular degeneration (AMD) is a leading cause of blindness worldwide, affecting approximately 200 million people. Geographic atrophy (GA), an advanced form of dry AMD, impacts over 5 million people globally and leads to progressive, irreversible vision loss. The economic burden of GA is substantial, with annual costs estimated at $39.4 billion in the United States alone. Despite its significant impact, there is limited awareness among stakeholders about the extent of GA's burden, resulting in inadequate research investment and few available treatment options. Current approaches to GA treatment aim to prevent and reduce the progression of existing atrophy, allowing individuals to extend their remaining vision considerably longer. However, there is no clear consensus on what constitutes a meaningful treatment outcome for patients, as perspectives may differ between patients, clinicians, regulatory bodies, and policymakers.Aims The aim of this study was to provide patient leaders' perspectives on the challenges faced by people living with GA and treatment expectations of current and potential therapies. The goal is to increase awareness of the disease burden and offer realistic treatment expectations of therapies from patients' viewpoints. The study employed a modified nominal group technique (NGT), a widely used validated consensus building method with five experienced patient leaders from 5 countries (Switzerland, Australia, South Africa, Spain and Brazil). All participants were associated with a membership organization of Retina International in their respective countries. The NGT process was partly conducted in-person at the 22nd Retina International World Congress, Dublin, Ireland and partly online.Findings The most significant challenges faced by people living with GA as ranked by the group include loss of independence, difficulty recognizing faces and impairment in daily living activities. Mental health issues such as depression and anxiety were also ranked high. Participants prioritised stability of vision, one time therapy and ability to recognise faces as their top expectations from GA treatments.Conclusion This study provides crucial insights into the multifaceted impact of Geographic Atrophy and patient expectations from treatments. The findings emphasize the need for a holistic approach to the management and care of GA that addresses not only clinical vision measures but also functional abilities, independence, and mental health. Moreover, the study demonstrated that the patients value stabilisation of vision as a meaningful treatment outcome and are keen to reduce the burden of frequent treatments by accepting one-time therapies such as gene therapy.Patient and service provider contribution This study sought the perspectives of experienced patient leaders. By focusing on these patient priorities, stakeholders can work towards developing more effective treatments, support systems, and policies that significantly improve the quality of life for individuals living with GA. This patient-centred perspective should guide future research, clinical practice, and policy decisions to better meet the needs of those affected by this sight-threatening condition.