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Proposal 1541

Title of the Proposed Research

Item calibration of the NEI VFQ-25 to improve future comparative research using patient-reported outcome (PRO) measures.

Lead Researcher

Judith E. Goldstein

Affiliation

Wilmer Eye Institute
Johns Hopkins School of Medicine

Funding Source

None; The costs of the analyses will be covered by funding at Johns Hopkins School of Medicine.

Potential Conflicts of Interest

None

Data Sharing Agreement Date

2 February 2017

Lay Summary

It is recognized in ophthalmology that changes in visual acuity do not entirely illustrate the effects of an intervention, and with increasing options for pharmaceutical therapy in retinal disorders, an understanding of the benefits from the patient perspective must be clearly understood.

Evaluation of visual ability, the ability to perform activities that depend on vision, can be made quantitative for measuring outcomes from the patient’s perspective by employing a visual function questionnaire (VFQ). Most VFQs have a fixed set of items that describe specific vision-dependent daily activities and ask the patient to rate the difficulty of performing those activities using an ordered set of response categories. The 25-item National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25), is one instrument used, however, it remains difficult to compare these findings across studies, as mean changes in raw scores are often published. The non-linearity of raw scores creates challenges to the meaningfulness of the findings, and raw scores are instrument-specific, which makes generalizing results difficult. The NEI VFQ-25, like most VFQ’s, permits opt out responses to irrelevant items (e.g., “not important”, etc.) which are scored as missing data. This reduces measurement precision, and depending on the estimation method employed, can distort the visual function measure. Rasch analysis can be employed to address both the issues of missing data and non-linearity of raw scores. This is best achieved by calibrating tem difficulty on the NEI VFQ-25. Item calibration then positions us to employ computer adaptive testing when administering the NEI VFQ-25.

Our aim to calibrate items on the NEI VFQ-25 involves assigning a difficulty estimate to those items that have a rating scale. Our group has performed this type of analysis with the Activity Inventory VFQ using 3200 low vision patients to calibrate items. To maximize precision of item calibrations, we will want to maximize the number of patient responses to the NEI VFQ-25. Our team has collected responses to the NEI VFQ-25 from approximately 1000 patients from low vision clinics across the U.S. With additional data from Genentech studies we can improve our precision of the estimates while at the same time ensuring that the item calibrations are relevant to patients participating in retinal-related clinical trials. We anticipate that this information may be of benefit to Genentech/Roche as it pursues various presentations and publications of its scientific data involving NEI VFQ-25 information, and may be of value in its indication pursuits with the EMA, FDA or other regulatory bodies that might be considering vision-related PRO’s. Once analysis is complete, our plan is to publish item calibrations in the ophthalmic literature to enhance future comparative research using publically available NEI VFQ-25 item calibrations.

Study Data Provided

ROCHE-FVF4170G: A Phase III, Double-Masked, Multicenter, Randomized, Sham Injection-Controlled Study Of The Efficacy And Safety Of Ranibizumab Injection In Subjects With Clinically Significant Macular Edema With Center Involvement Secondary To Diabetes Mellitu
ROCHE-FVF4168G: A Phase III, Double-Masked, Multicenter, Randomized, Sham Injection-Controlled Study Of The Efficacy And Safety Of Ranibizumab Injection In Subjects With Clinically Significant Macular Edema With Center Involvement Secondary To Diabetes Mellitus
ROCHE-FVF4579G: A Phase III, Double-masked, Multicenter, Randomized, Active Treatment-controlled Study of the Efficacy and Safety of 0.5 mg and 2.0 mg Ranibizumab Administered Monthly or on an As-needed Basis (PRN) in Patients With Subfoveal Neovascular Age-related Macular Degeneration
ROCHE-FVF2598G: A Phase IIIb, Multicenter, Randomized, Double Masked, SHAM Injection, Controlled Study of the Efficacy and Safety of Ranibizumab in Subjects with Subfoveal Choroidal Neovascularization (CNV) with or without Classic CNV Secondary to Age Related Macular Degeneration
ROCHE-FVF3192G: An open-label, multicenter Extension study to evaluate the safety and tolerability of RANIBIZUMAB in subjects with choroidal neovascularization (CNV) secondary to age related macular degeneration (AMD) or macular edema secondary to RETINAL VEIN OCCLUSION (RVO) who have completed a genentech sponsored RANIBIZUMAB STUDY
ROCHE-FVF3426G: An open-label, multicenter Extension study to evaluate the safety and tolerability of RANIBIZUMAB in subjects with choroidal neovascularization (CNV) secondary to age related macular degeneration (AMD) or macular edema secondary to RETINAL VEIN OCCLUSION (RVO) who have completed a genentech sponsored RANIBIZUMAB STUDY-
ROCHE-FVF3689G: A Phase IIIb, Single-Masked, Multicenter, Randomized Study to Evaluate the Safety and Tolerability of Ranibizumab in Naive and Previously Treated Subjects With Choroidal Neovascularization (CNV) Secondary to Age-Related Macular Degeneration (AMD)-
ROCHE-FVF4165G: A Phase III, Multicenter, Randomized, Sham Injection Controlled Study of the Efficacy and Safety of Ranibizumab Injection Compared with Sham in Subjects with Macular Edema Secondary to Branch Retinal Vein Occlusion
ROCHE-FVF4166G: A PHASE III, MULTICENTER, RANDOMIZED, sham CONTROLLED STUDY OF THE efficacy and SAFETY OF RANIBIZUMAB compared to Sham IN SUBJECTS WITH macular edema secondary to central retinal vein occlusion
ROCHE-FVF2587G: A Phase III, Placebo-Controlled, Multi-Center Study to Examine theSafety and Efficacy of Multiple-Dose Intravitreal Injections of rhuFabV2 in Subjects with Age-Related Macular Degeneration who have Predominant Classic Lesion Degeneration
ROCHE-FVF4967G: A Study Evaluating Dosing Regimens for Treatment With Intravitreal Ranibizumab Injections in Subjects With Macular Edema Following Retinal Vein Occlusion

Statistical Analysis Plan

Publication Citation

The publication citation will be added after the research is published.

Summary Results

Results summary or link will be posted when available.