Best Practices for the Design of Clinical Trials Related to the Visual System

Literature Cited

1. Bailey IL, Jackson AJ, Minto H, Greer RB, Chu MA. 2012. The Berkeley rudimentary vision test. Optom. Vis. Sci. 89:1257–64
   [Google Scholar]
2. Balan TA, Putter H. 2020. A tutorial on frailty models. Stat. Methods Med. Res. 29:3424–54
   [Google Scholar]
3. Beck RW, Maguire MG, Glassman A, Ferris FL, Lindblad A, Bressler NM. 2007. Visual acuity as an outcome measure in clinical trials of retinal diseases. Ophthalmology 114:1804–9
   [Google Scholar]
4. Beck RW, Moke PS, Turpin AH, Ferris FL III, SanGiovanni JP et al. 2003. A computerized method of visual acuity testing: adaptation of the Early Treatment of Diabetic Retinopathy Study testing protocol. Am. J. Ophthalmol. 135:194–205
   [Google Scholar]
5. Brown DM, Kaiser PK, Michels M, Soubrane G, Heier JS et al. 2006. Ranibizumab versus verteporfin for neovascular age-related macular degeneration. N. Engl. J. Med. 355:1432–44
   [Google Scholar]
6. CATT Res. Group 2011. Ranibizumab and bevacizumab for neovascular age-related macular degeneration. N. Engl. J. Med. 364:1897–908
   [Google Scholar]
7. Cent. Dis. Control Prev 2020. National Diabetes Statistics Report, 2020 Atlanta, GA: Cent. Dis. Control Prev.
   [Google Scholar]
8. Chew EY, Clemons T, SanGiovanni JP, Danis R, Domalpally A et al. 2012. The Age-Related Eye Disease Study 2 (AREDS2): study design and baseline characteristics (AREDS2 report number 1). Ophthalmology 119:2282–89
   [Google Scholar]
9. Consol. Stand. Rep. Trials Group 2020. Extensions of the CONSORT Statement Rep., Consol. Stand. Rep. Trials Group Ottawa, Can: http://www.consort-statement.org/extensions
   [Google Scholar]
10. Diabet. Retin. Study Res. Group 1976. Preliminary report on effects of photocoagulation therapy. Am. J. Ophthalmol 81:383–97
    [Google Scholar]
11. Dry Eye Assess. Manag. (DREAM) Study Res. Group 2018. n-3 fatty acid supplementation for treatment of dry eye disease. N. Engl. J. Med 378:1681–90
    [Google Scholar]
12. Eapen ZJ, Lauer MS, Temple RJ. 2014. The imperative of overcoming barriers to the conduct of large, simple trials. JAMA 311:1397–98
    [Google Scholar]
13. Early Treat. Diabet. Retin. Study Res. Group 1991. Grading diabetic retinopathy from stereoscopic color fundus photographs—an extension of the modified Airlie House classification. ETDRS report number 10 Ophthalmology 98:786–806
    [Google Scholar]
14. Ferris FL, Kassoff A, Bresnick GH, Bailey I. 1982. New visual acuity charts for clinical research. Am. J. Ophthalmol. 94:91–96
    [Google Scholar]
15. Flight L, Julious SA. 2016. Practical guide to sample size calculations: non-inferiority and equivalence trials. Pharm. Stat. 15:80–89
    [Google Scholar]
16. Friedman LM, Furberg CD, Demets DL, Reboussin DM, Granger CB. 2015. Fundamentals of Clinical Trials Berlin: Springer, 5th ed..
    [Google Scholar]
17. Gauderman WJ, Barlow WE. 1992. Sample size calculations for ophthalmologic studies. Arch. Ophthalmol. 110:690–92
    [Google Scholar]
18. Gazzard G, Konstantakopoulou E, Garway-Heath D, Barton K, Wormald R et al. 2018. Laser in Glaucoma and Ocular Hypertension (LiGHT) trial. A multicentre, randomised controlled trial: design and methodology. Br. J. Ophthalmol. 102:593–98
    [Google Scholar]
19. Glaucoma Laser Trial Res. Group 1991. The Glaucoma Laser Trial (GLT): 4. Contralateral effects of timolol on the intraocular pressure of eyes treated with ALT. Ophthalmic Surg 22:324–29
    [Google Scholar]
20. Guymer RH, Markey CM, McAllister IL, Gillies MC, Hunyor AP et al. 2019. Tolerating subretinal fluid in neovascular age-related macular degeneration treated with ranibizumab using a treat-and-extend regimen. FLUID study 24-month results. Ophthalmology 126:723–34
    [Google Scholar]
21. Hallstrom A, Davis K. 1988. Imbalance in treatment assignments in stratified blocked randomization. Control Clin. Trials 9:375–82
    [Google Scholar]
22. Hulley SB, Cummings SR, Browner WS, Grady DG, Newman TB. 2013. Designing Clinical Research Philadelphia: Lippincott Williams & Wilkins, 4th ed..
    [Google Scholar]
23. Jaffe GJ, Martin DF, Toth CA, Daniel E, Maguire MG et al. 2013. Macular morphology and visual acuity in the Comparison of Age-Related Macular Degeneration Treatments Trials (CATT). Ophthalmology 120:1860–70
    [Google Scholar]
24. Kim BJ, Hunter A, Brucker AJ, Hahn P, Gehrs K et al. 2020. Evaluation of orally administered alpha lipoic acid as a treatment for geographic atrophy. Ophthalmol. Retina 4:889–98
    [Google Scholar]
25. Kinsey VE, Jacobus JT, Hemphill FM. 1956. Retrolental fibroplasia: cooperative study of retrolental fibroplasia and the use of oxygen. AMA Arch. Ophthalmol. 56:481–543
    [Google Scholar]
26. Krummenauer F, Dick B, Schwenn O, Pfeiffer N. 2002. The determination of sample size in controlled clinical trials in ophthalmology. Br. J. Ophthalmol. 86:946–47
    [Google Scholar]
27. Ledolter J, Gramlich OW, Kardon RH. 2020. Parametric statistical inference for comparing means and variances. Investig. Ophthalmol. Vis. Sci. 61:825
    [Google Scholar]
28. Ledolter J, Kardon RH. 2020. Focus on data: statistical design of experiments and sample size selection using power analysis. Investig. Ophthalmol. Vis. 61:811
    [Google Scholar]
29. Lin DY, Wei LJ. 1989. The robust inference for the proportional hazards model. J. Am. Stat. Assoc. 84:1074–78
    [Google Scholar]
30. Mo Y, Lim C, Watson JA, White NJ, Cooper BS. 2020. Non-adherence in non-inferiority trials: pitfalls and recommendations. BMJ 370:m2215
    [Google Scholar]
31. Multicent. Uveitis Steroid Treat. (MUST) Trial Res. Group Kempen JH, Altaweel MM, Holbrook JT, Jabs DA et al. 2011. Randomized comparison of systemic anti-inflammatory therapy versus fluocinolone acetonide implant for intermediate, posterior, and panuveitis: the multicenter uveitis steroid treatment trial. Ophthalmology 118:1916–26
    [Google Scholar]
32. Natl. Inst. Health 2003. Final NIH statement on sharing research data Rep. NOT-OD-03-032, Natl. Inst. Health Bethesda, MD:
    [Google Scholar]
33. Natl. Inst. Health 2020. Final NIH policy for data management and sharing Rep. NOT-OD-21-013, Natl. Inst. Health Bethesda, MD:
    [Google Scholar]
34. Patz A, Hoeck LE, de la Cruz D. 1952. Studies on the effect of high oxygen administration in retrolental fibroplasia. I. Nursery observations. Am. J. Ophthalmol. 35:1248–53
    [Google Scholar]
35. Piantadosi S. 2017. Clinical Trials: A Methodologic Perspective Hoboken, NJ: Wiley, 3rd ed..
    [Google Scholar]
36. Rosenfeld PJ, Brown DM, Heier JS, Boyer DS, Kaiser PK et al. 2006. Ranibizumab for neovascular age-related macular degeneration. N. Engl. J. Med. 355:1419–31
    [Google Scholar]
37. Saldanha IJ, Lidsley K, Do DV, Chuck RS, Meyerle C et al. 2017. Comparison of clinical trial and systematic review outcomes for the 4 most prevalent eye diseases. JAMA Ophthalmol 135:933–40
    [Google Scholar]
38. Sanchez MM, Chen X. 2006. Choosing the analysis population in non-inferiority studies: per protocol or intent-to-treat. Stat. Med. 25:1169–81
    [Google Scholar]
39. Schmidt-Erfurth U, Waldstein SM, Deak G, Kundi M, Simader C. 2015. Pigment epithelial detachment followed by retinal cystoid degeneration leads to vision loss in treatment of neovascular age-related macular degeneration. Ophthalmology 122:822–32
    [Google Scholar]
40. Schulz KF. 1995. Subverting randomization in controlled trials. JAMA 274:1456–58
    [Google Scholar]
41. Schulz KF, Altman DG, Moher DCONSORT Group 2010. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. J. Clin. Epidemiol. 63:834–40
    [Google Scholar]
42. Sedgwick P. 2015. Intention to treat analysis versus per protocol analysis of trial data. BMJ 350:h681
    [Google Scholar]
43. Seitzman GD, Lietman TM. 2019. Dry eye research—still regressing?. Ophthalmology 126:192–94
    [Google Scholar]
44. Tavel ME. 2014. The placebo effect: the good, the bad, and the ugly. Am. J. Med. 127:484–88
    [Google Scholar]
45. Treat. Age-Relat. Macular Degener. Photodyn. Ther. (TAP) Study Group 1999. Photodynamic therapy of subfoveal choroidal neovascularization in age-related macular degeneration with verteporfin: one-year results of 2 randomized clinical trials—TAP report 1. Arch. Ophthalmol. 117:1329–45
    [Google Scholar]
46. U.S. Food Drug Admin 2016. FDA draft guidance for industry: non-inferiority clinical trials to establish effectiveness Rep., U.S. Food Drug Admin Washington, DC:
    [Google Scholar]
47. U.S. Food Drug Admin 2020. The drug development process. Step 3: clinical research Rep., U.S. Food Drug Admin Washington, DC: https://www.fda.gov/patients/drug-development-process/step-3-clinical-research
    [Google Scholar]
48. World Health Organ 2020. Clinical trials Q&A. Rep., World Health Organ Geneva: https://www.who.int/news-room/q-a-detail/clinical-trials
    [Google Scholar]
49. Yeh S, Khurana RN, Shah M, Henry CR, Wang RC et al. 2020. Efficacy and safety of suprachoroidal CLS-TA for macular edema secondary to noninfectious uveitis: phase 3 randomized trial. Ophthalmology 127:948–55
    [Google Scholar]
50. Ying GS, Maguire MG, Glynn R, Rosner B. 2017. Tutorial on biostatistics: linear regression analysis of continuous correlated eye data. Ophthalmic Epidemiol 24:130–40
    [Google Scholar]
51. Ying GS, Maguire MG, Glynn R, Rosner B 2018. Tutorial on biostatistics: statistical analysis for correlated binary eye data. Ophthalmic Epidemiol 25:1–12
    [Google Scholar]