Despite significant advances in the treatment and understanding of late stage age-related macular degeneration (AMD), it continues to be the main cause of irreversible severe visual loss in Europe and its prevalence and incidence will increase with current demographic trends. In order to reduce the significant burden of late stage AMD, novel interventions should aim at stopping or delaying progression from the preceding disease stage intermediate AMD (iAMD) to late stage AMD. As a prerequisite, validated clinical endpoints for iAMD are needed. These should be acceptable to regulatory agencies, health technology assessment (HTA) bodies, and payers. Currently such endpoints do not exist for iAMD clinical trials (CTs). In addition, there is good evidence indicating that patients with iAMD experience some impairment of visual function yet it is unknown to what extend this impacts the patients’ life nor can it be reliably measured and quantified. It is also unknown whether there are specific risk factors in the population of iAMD patients which identify those with more rapid progression to late stages of the disease. Therefore, to enable successful development of iAMD interventions validated functional, morphological and patient--reported endpoints for CTs, which are clinically meaningful and accepted by regulatory agencies, are required. In addition, functional decline in iAMD, as well as, specific risk factors for iAMD progression to late stage AMD need to be better characterized to inform and improve conduct of future iAMD CTs. Against this background, the major objective of MACUSTAR is to develop novel clinical endpoints for CTs with a regulatory and patient access intention in patients with iAMD. Additional objectives are to characterize visual impairment in iAMD and its progression, as well as, identify risk factors for progression. For clinical endpoint development, functional, structural and patient-reported outcome measures will be assessed with regards to

Since the establishment of EATRIS as an ERIC in 2014, the infrastructure has developed a growing portfolio of services and collaborative projects serving several user groups including industry (large pharma and SME), academia, research funders and patients. EATRIS has progressed from proposing a strong proof of concept service portfolio, to building and delivering innovative tools for translational research and is now advancing its strategy to further exploit these tools for the extended research and innovation community. EATRIS-CONNECT will accelerate this strategy by focusing specifically on advancing the digital readiness of EATRIS to support biomedical challenges in Europe and provide translational solutions for the benefit of personalised medicine. Through curated access to novel digital tools and services, as well as node strengthening, EATRIS-CONNECT will equip EATRIS across the entire distributed research infrastructure to address bottlenecks and challenges for the application of digital solutions in the context of use. Moreover, the project will ensure this digital transformation is environmentally sound by improving energy, resource, and material efficiency, supporting the environmental goals of the European Green Deal. The project will promote the cross-border alignment in the adoption and application of digital tools and help consolidate the European RI landscape by fostering interdisciplinary collaborations between cross-sector RI and exploring how RI can together unlock innovation in the digital domain. EATRIS-CONNECT will provide the pathway for the evolution of EATRIS. All 14 national EATRIS nodes form the central pillar of EATRIS-CONNECT, facilitating the alignment of strategic national priorities across the infrastructure and empowering EATRIS widening nodes to strengthen their digital competencies, ultimately ensuring the long-term sustainability of EATRIS.