Bonini, S., Rama, P., Olzi, D., & Lambiase, A. (2003). Neurotrophic keratitis. Eye (London, England), 17(8), 989–995. https://doi.org/10.1038/sj.eye.6700616.
Article
CAS
Google Scholar
Dua, H. S., Said, D. G., Messmer, E. M., Rolando, M., Benitez-Del-Castillo, J. M., Hossain, P. N., et al. (2018). Neurotrophic keratopathy. Progress in Retinal and Eye Research. https://doi.org/10.1016/j.preteyeres.2018.04.003.
Sacchetti, M., & Lambiase, A. (2014). Diagnosis and management of neurotrophic keratitis. Clinical Ophthalmology, 8, 571–579. https://doi.org/10.2147/OPTH.S45921.
Article
PubMed
Google Scholar
Gross, E. B. (1997). Neurotrophic keratitis. In J. Krachmer, M. Mannis, & E. Holland (Eds.), Cornea (3rd ed., p. 1340). St. Louis: Mosby.
Google Scholar
Mackie, I. A. (1995). Neuroparalytic keratitis. In F. Fraunfelder, F. Roy, & S. Meyer (Eds.), Current ocular therapy. Philadelphia: WB Saunders.
Google Scholar
Lanigan, D. T., Romanchuk, K., & Olson, C. K. (1993). Ophthalmic complications associated with orthognathic surgery. Journal of Oral and Maxillofacial Surgery, 51(5), 480–494.
Article
CAS
Google Scholar
Hyndiuk, R. A., Kazarian, E. L., Schultz, R. O., & Seideman, S. (1977). Neurotrophic corneal ulcers in diabetes mellitus. Archives of Ophthalmology, 95(12), 2193–2196.
Article
CAS
Google Scholar
Semeraro, F., Forbice, E., Romano, V., Angi, M., Romano, M. R., Filippelli, M. E., et al. (2014). Neurotrophic keratitis. Ophthalmologica., 231(4), 191–197. https://doi.org/10.1159/000354380.
Article
CAS
PubMed
Google Scholar
Faulkner, W., & Varley, G. (1997). Corneal diagnostic technique. In J. Krachmer, M. Mannis, & E. Holland (Eds.), Cornea (3rd ed., p. 1340). St. Louis: Mosby.
Google Scholar
NCT01756456: Evaluation of Safety and Efficacy of rhNGF in Patients With Stage 2 and 3 Neurotrophic Keratitis. (REPARO) [database on the Internet]. National Library of Medicine. 2012a. Available from: https://clinicaltrials.gov/ct2/show/study/NCT01756456. Accessed 15 July 2019.
NCT02227147: Evaluation of Efficacy of 20 μg/ml rhNGF New Formulation (With Anti-oxidant) in Patients With Stage 2 and 3 NK [database on the Internet]. National Library of Medicine. 2012b. Available from: https://clinicaltrials.gov/ct2/show/NCT02227147?term=NGF0214&rank=1. Accessed: 15 July 2019.
Bonini, S., Lambiase, A., Rama, P., Sinigaglia, F., Allegretti, M., Chao, W., et al. (2018b). Phase II randomized, double-masked, vehicle-controlled trial of recombinant human nerve growth factor for neurotrophic keratitis. Ophthalmology., 125(9), 1332–1343. https://doi.org/10.1016/j.ophtha.2018.02.022.
Article
PubMed
Google Scholar
European Medicines Agency, Committee for Medicinal Products for Human Use (CHMP), Efficacy Working Party (EWP). (2005). Reflection paper on the regulatory guidance for the use of health-related quality of life (HRQL) measures in the evaluation of medicinal products
http://www.emea.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003637.pdf.
Google Scholar
U.S. Food and Drug Administration. (2009). Guidance for industry. Patient-reported outcome measures: Use in medical product development to support labeling claims. Rockville: https://www.fda.gov/downloads/drugs/guidances/ucm193282.pdf.
DeMuro, C., Clark, M., Doward, L., Evans, E., Mordin, M., & Gnanasakthy, A. (2013). Assessment of PRO label claims granted by the FDA as compared to the EMA (2006-2010). Value in Health, 16(8), 1150–1155. https://doi.org/10.1016/j.jval.2013.08.2293.
Article
PubMed
Google Scholar
U.S. Food and Drug Administration. (2015). Roadmap to patient-focused outcome measurement in clinical trials. Rockville: https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm370177.htm.
Patrick, D. L., Burke, L. B., Gwaltney, C. J., Leidy, N. K., Martin, M. L., Molsen, E., et al. (2011a). Content validity--establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part 1--eliciting concepts for a new PRO instrument. Value in Health, 14(8), 967–977. https://doi.org/10.1016/j.jval.2011.06.014.
Article
PubMed
Google Scholar
Patrick, D. L., Burke, L. B., Gwaltney, C. J., Leidy, N. K., Martin, M. L., Molsen, E., et al. (2011b). Content validity--establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO good research practices task force report: Part 2--assessing respondent understanding. Value in Health, 14(8), 978–988. https://doi.org/10.1016/j.jval.2011.06.013.
Article
PubMed
Google Scholar
Mantelli, F., et al. (2015). Congenital corneal anesthesia and neurotrophic keratitis: Diagnosis and management. BioMed Research International, 2015, 805876.
Article
Google Scholar
Venkateswaran, N., Yeaney, G., Chung, M., & Hindman, H. B. (2014). Recurrent nontuberculous mycobacterial endophthalmitis: A diagnostic conundrum. Clinical Ophthalmology, 8, 837–842. https://doi.org/10.2147/OPTH.S56670.
Article
PubMed
Google Scholar
Carta, A., Braccio, L., Belpoliti, M., Soliani, L., Sartore, F., Gandolfi, S. A., et al. (1998). Self-assessment of the quality of vision: Association of questionnaire score with objective clinical tests. Current Eye Research, 17(5), 506–511.
Article
CAS
Google Scholar
Mangione, C. M., Lee, P. P., Gutierrez, P. R., Spritzer, K., Berry, S., Hays, R. D., et al. (2001). Development of the 25-item national eye institute visual function questionnaire. Archives of Ophthalmology, 119(7), 1050–1058.
Article
CAS
Google Scholar
Schaumberg, D. A., Gulati, A., Mathers, W. D., Clinch, T., Lemp, M. A., Nelson, J. D., et al. (2007). Development and validation of a short global dry eye symptom index. The Ocular Surface, 5(1), 50–57.
Article
Google Scholar
Walt, J. G., Rowe, M. M., Stern, K. L. (1997). Evaluating the functional impact of dry eye: the Ocular Surface Disease Index [abstract]. Drug information journal, 311436.
Weih, L. M., Hassell, J. B., & Keeffe, J. (2002). Assessment of the impact of vision impairment. Investigative Ophthalmology & Visual Science, 43(4), 927–935.
Google Scholar
Wolffsohn, J. S., & Cochrane, A. L. (2000). Design of the low vision quality-of-life questionnaire (LVQOL) and measuring the outcome of low-vision rehabilitation. American Journal of Ophthalmology, 130(6), 793–802.
Article
CAS
Google Scholar
Benjamin, K., Vernon, M. K., Patrick, D. L., Perfetto, E., Nestler-Parr, S., & Burke, L. (2017). Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: An ISPOR COA emerging good practices task force report. Value in Health, 20(7), 838–855. https://doi.org/10.1016/j.jval.2017.05.015.
Article
PubMed
Google Scholar
U.S. Food and Drug Administration. (2018). Guidance for industry: Rare diseases—Early drug development and the role of pre-IND meetings. Rockville: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-diseases-early-drug-development-and-role-pre-ind-meetings. Accessed 15 Feb 2019.
Mangione, C. (2000). 25-item national eye institute visual function questionnaire (VFQ-25). Bethesda: National Eye Institute https://nei.nih.gov/sites/default/files/nei-pdfs/manual_cm2000.pdf. Accessed 15 Feb 2019.
Google Scholar
Friese, S., & Ringmayr, T. G. (2014). ATLAS. Ti user guide and reference. Berlin: ATLAS.ti Scientific Software Development GmBH.
Google Scholar
Leidy, N. K., & Vernon, M. (2008). Perspectives on patient-reported outcomes : Content validity and qualitative research in a changing clinical trial environment. Pharmacoeconomics., 26(5), 363–370.
Article
Google Scholar
H.R.6—114th Congress 21st Century Cures Act Subtitle A – Patient-Focused Drug Development (Sec.2001). 2015–2016.
Perfetto, E. M., Burke, L., Oehrlein, E. M., & Epstein, R. S. (2015). Patient-focused drug development: A new direction for collaboration. Medical Care, 53(1), 9–17. https://doi.org/10.1097/MLR.0000000000000273.
Article
PubMed
Google Scholar
Selby, J. V., Beal, A. C., & Frank, L. (2012). The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda. JAMA., 307(15), 1583–1584. https://doi.org/10.1001/jama.2012.500.
Article
CAS
PubMed
Google Scholar
Snyder, C. F., Aaronson, N. K., Choucair, A. K., Elliott, T. E., Greenhalgh, J., Halyard, M. Y., et al. (2012). Implementing patient-reported outcomes assessment in clinical practice: A review of the options and considerations. Quality of Life Research, 21(8), 1305–1314. https://doi.org/10.1007/s11136-011-0054-x.
Article
PubMed
Google Scholar
European Organisation for Rare Disorders. (2009). The voice of 12,000 patients: Experiences and expectations of rare disease patients on diagnosis and Care in Europe.
Google Scholar
European Organisation for Rare Disorders. (2011). October position paper: Patients’ priorities and needs for RD research 2014-2020.
Google Scholar
Suner, I. J., Kokame, G. T., Yu, E., Ward, J., Dolan, C., & Bressler, N. M. (2009). Responsiveness of NEI VFQ-25 to changes in visual acuity in neovascular AMD: Validation studies from two phase 3 clinical trials. Investigative Ophthalmology & Visual Science, 50(8), 3629–3635.
Article
Google Scholar
Orr, P., Rentz, A. M., Margolis, M. K., Revicki, D. A., Dolan, C. M., Colman, S., et al. (2011). Validation of the national eye institute visual function Questionnaire-25 (NEI VFQ-25) in age-related macular degeneration. Investigative Ophthalmology & Visual Science, 52(6), 3354–3359. https://doi.org/10.1167/iovs.10-5645.
Article
Google Scholar
Sasinowski, F. (2011). Quantum of effectiveness evidence in FDA’s approval of orphan drugs (cataloguing FDA’s flexibility in regulating therapies for persons with rare disorders). NORD., 238–263.