The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease
Author:
Funder
University of Toronto and University Health Network
Hospital for Sick Children
Foundation Fighting Blindness
Publisher
Springer Science and Business Media LLC
Subject
Ophthalmology
Link
https://link.springer.com/content/pdf/10.1007/s40123-022-00514-x.pdf
Reference43 articles.
1. Gong J, Cheung S, Fasso-Opie A, et al. The impact of inherited retinal diseases in the United States of America (US) and Canada from a cost-of-illness perspective. Clin Ophthalmol. 2021;15:2855–66. https://doi.org/10.2147/OPTH.S313719.
2. Heon E, Koenekoop R. Treatments for inherited retinal degenerations are coming to Canada: brief update on a new standard of care for inherited retinal degenerations. Can J Ophthalmol. 2021;56:e34–5. https://doi.org/10.1016/j.jcjo.2020.10.019.
3. US Department of Health and Human Services FDA Center for Drug Evaluation and Research, US Department of Health and Human Services FDA Center for Biologics Evaluation and Research, US Department of Health and Human Services FDA Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance. Health Qual Life Outcomes. 2006;4:79. https://doi.org/10.1186/1477-7525-4-79.
4. Patrick DL, Burke LB, Powers JH, et al. Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health. 2007;10:S125–37. https://doi.org/10.1111/j.1524-4733.2007.00275.x.
5. Lacy GD, Abalem MF, Musch DC, et al. Patient-reported outcome measures in inherited retinal degeneration gene therapy trials. Ophthalmic Genet. 2020;41:1–6. https://doi.org/10.1080/13816810.2020.1731836.
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