June 2020
Volume 61, Issue 7
ARVO Annual Meeting Abstract  |   June 2020
Accelerating research in rare inherited retinal disorders (IRDs) through an international consortium
Author Affiliations & Notes
  • Janet K Cheetham
    Celeris Consulting, Laguna Niguel, California, United States
  • Allison Ayala
    Jaeb Center for Health Research, Florida, United States
  • David G Birch
    Retina Foundation of the Southwest, Texas, United States
  • Jacque L Duncan
    University of California, San Francisco, California, United States
  • Todd Durham
    Foundation Fighting Blindness, Maryland, United States
  • Frederick L Ferris
    Ophthalmic Research Consultants, North Carolina, United States
  • Maureen Maguire
    University of Pennsylvania, Pennsylvania, United States
  • Mark E Pennesi
    Casey Eye Institute - OSHU, Oregon, United States
  • Footnotes
    Commercial Relationships   Janet Cheetham, None; Allison Ayala, None; David Birch, None; Jacque Duncan, None; Todd Durham, None; Frederick Ferris, None; Maureen Maguire, None; Mark Pennesi, None
  • Footnotes
    Support  Foundation Fighting Blindness
Investigative Ophthalmology & Visual Science June 2020, Vol.61, 3356. doi:
  • Views
  • Share
  • Tools
    • Alerts
      This feature is available to authenticated users only.
      Sign In or Create an Account ×
    • Get Citation

      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.

      Download citation file:

      © ARVO (1962-2015); The Authors (2016-present)

  • Supplements

Purpose : 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.

Methods : 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.

Results : 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.

Conclusions : 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.


This PDF is available to Subscribers Only

Sign in or purchase a subscription to access this content. ×

You must be signed into an individual account to use this feature.