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Janet K Cheetham, Allison Ayala, David G Birch, Jacque L Duncan, Todd Durham, Frederick L Ferris, Maureen Maguire, Mark E Pennesi; Accelerating research in rare inherited retinal disorders (IRDs) through an international consortium. Invest. Ophthalmol. Vis. Sci. 2020;61(7):3356.
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© ARVO (1962-2015); The Authors (2016-present)
Following the success of the DRCR Retina Network model, the Foundation Fighting Blindness (FFB) sought to build a Consortium of clinical centers to conduct research in patients with rare inherited retinal disorders (IRDs), with the goal to accelerate development of treatments for IRDs.
In 2016, an Executive Committee (EC) was formed to facilitate the FFB mission to develop a clinical research Consortium. The plan for the Consortium model includes the following:(1) Investigators from participating clinical centers collaborate on ideas for hypotheses, study designs, and publications.(2) Consortium studies provide researchers and clinicians with long-term data about disease onset and progression and identify sensitive structural and functional outcome measures of treatment effects for planning future clinical trials.(3) Data from completed studies will be archived in an open central repository to stimulate further hypothesis generation and innovation.
Since the FFB Consortium inception, a coordinating center, two reading centers, and 147 investigators at 38 sites (28 US and 10 international) have collaborated with FFB and the Consortium EC to develop a research infrastructure. Three prospective studies have been launched, to date. The inaugural study, Rate of Progression in USH2A-related Retinal Degenerations (RUSH2A), is a 4-year natural history study that concluded recruitment of over 120 participants in early 2019. A second 4-year natural history study, the Rate of Progression in EYS Related Retinal Degeneration (Pro-EYS), was launched in 2019 and is expected to recruit 100 participants by 2020. A third study to develop and validate a new patient-reported outcomes measure for use in patients with retinitis pigmentosa was also launched in 2019.
The FFB Consortium has developed an efficient network of centers and collaborators, capable of conducting large multi-center studies that follow standards of Good Clinical Practice. The findings from the Consortium studies will be published and widely disseminated so that we can share what we’ve learned with therapy developers around the world. Information about the Consortium, listings of participating centers, and publications can be found on ffb.jaeb.org.
This is a 2020 ARVO Annual Meeting abstract.
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