Safety and efficacy of ixoberogene soroparvovec in neovascular age-related macular degeneration in the United States (OPTIC): a prospective, two-year, multicentre phase 1 study.

Background: Gene therapy, successfully used in rare, monogenetic disorders, may prove to be a durable management approach for common, polygenetic conditions, including neovascular age-related macular degeneration (nAMD). Repeated injections, oftentimes monthly, and possibly for decades, of vascular endothelial growth factor antagonists (anti-VEGF), is the standard for nAMD. We hypothesised that an in-office, intravitreal administration of ixoberogene soroparvovec (ixo-vec, formerly ADVM-022), a single-dose gene therapy encoding for the proven anti-VEGF protein, aflibercept, would transform retinal cells to continually produce aflibercept to minimise treatment burden in nAMD.

Endophthalmitis rates among patients receiving intravitreal anti-VEGF injections: a USA claims analysis.

Purpose: Intravitreal (IVT) injections of the anti-vascular endothelial growth factor (VEGF) agents aflibercept, bevacizumab, and ranibizumab are commonly prescribed to treat neovascular age-related macular degeneration (nAMD). Studies comparing inflammation rates in large populations of patients receiving these agents and the treatment of ocular inflammation post-IVT anti-VEGF injections are scarce. In this study, we compared rates of endophthalmitis claims (sterile and infectious) following IVT anti-VEGF injections to determine the risk factors associated with developing endophthalmitis, and examined the claims for subsequent treatment.

Adherence to the American Diabetes Association retinal screening guidelines for population with diabetes in the United States.

Purpose: (1) To assess long-term adherence to American Diabetes Association guideline-recommended retinal screening among population with diabetes in the United States. (2) To determine factors associated with long-term adherence to routine eye screening exams.

Methods: A retrospective cohort study was conducted in adult patients with diabetes identified from January 2009 to December 2010.

The comparative effectiveness and cost-effectiveness of ranibizumab for neovascular macular degeneration revisited.

Background: To compare a near decade of follow-up, newer control cohort data, use of both the societal and third party insurer cost perspectives, and integration of unilateral/bilateral therapy on the comparative effectiveness and cost-effectiveness of intravitreal ranibizumab therapy for neovascular, age-related macular degeneration (AMD).

Methods: Value-Based Medicine, 12-year, combined-eye model, cost-utility analysis employing MARINA and HORIZON clinical trial data. Preference-based comparative effectiveness outcomes were quantified in (1) QALY (quality-adjusted life-year) gain, and (2) percent improvement in quality-of-life, while cost-effectiveness outcomes were quantified in (3) the cost-utility ratio (CUR) and financial return-on-investment (ROI) to society.

The Burden of Retinal Venous Occlusion: An Assessment of Fellow Eyes in 1000 Cases.

Objective: To assess retinal vein occlusion (RVO) clinical features to create a simulation model quantifying the preference-based, patient value gain (benefit) and cost-utility (cost-effectiveness) of RVO therapy.

Design: Retrospective analysis data integrated with patient utilities and an ocular cost-utility model for RVO.

Participants: One thousand consecutive Wills Eye Hospital Retina Service RVO patients seen from January 2010 through April 2011.

RESPONSIVENESS OF THE NATIONAL EYE INSTITUTE VISUAL FUNCTION QUESTIONNAIRE-25 TO VISUAL ACUITY GAINS IN PATIENTS WITH DIABETIC MACULAR EDEMA: Evidence From the RIDE and RISE Trials.

Purpose: To evaluate the responsiveness of the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) in patients with diabetic macular edema using data from the RIDE and RISE trials.

Methods: Patients were randomized to monthly intravitreal ranibizumab 0.3 mg, 0.

Effect of Ranibizumab on the Decision to Drive and Vision Function Relevant to Driving in Patients With Diabetic Macular Edema: Report From RESTORE, RIDE, and RISE Trials.

Importance: The potential effect of treatments for diabetic macular edema (DME) on driving should be of value to patients and clinicians, such as ophthalmologists and other physicians, who treat patients with diabetes mellitus.

Objective: To determine the effect of ranibizumab on driving and patient-reported vision function relevant to driving among patients with DME.

Design, Setting, And Participants: This exploratory post hoc analysis was conducted between October 1, 2011, and July 25, 2015, based on deidentified data from phase 3, multicenter, randomized clinical trials (RIDE, RISE, and RESTORE trials).

Longitudinal Commercial Claims-Based Cost Analysis of Diabetic Retinopathy Screening Patterns.

Background: Diabetic retinopathy is one of the most common complications of diabetes. The screening of patients with diabetes to detect retinopathy is recommended by several professional guidelines but is an underutilized service.

Objective: To analyze the relationship between the frequency of retinopathy screening and the cost of care in adult patients with diabetes.

SOCIETAL COSTS ASSOCIATED WITH NEOVASCULAR AGE-RELATED MACULAR DEGENERATION IN THE UNITED STATES.

Purpose: The purpose of this study was to use a cross-sectional prevalence-based health care economic survey to ascertain the annual, incremental, societal ophthalmic costs associated with neovascular age-related macular degeneration.

Methods: Consecutive patients (n = 200) with neovascular age-related macular degeneration were studied. A Control Cohort included patients with good (20/20-20/25) vision, while Study Cohort vision levels included Subcohort 1: 20/30 to 20/50, Subcohort 2: 20/60 to 20/100, Subcohort 3: 20/200 to 20/400, and Subcohort 4: 20/800 to no light perception.

The Cost-Effectiveness of Ranibizumab for the Treatment of Diabetic Macular Edema.

Purpose: To assess the incremental, comparative effectiveness (patient value gain) and cost effectiveness (financial value gain) associated with 0.3-mg intravitreal ranibizumab injection therapy versus sham therapy for diabetic macular edema (DME).

Design: Value-Based Medicine (Center for Value-Based Medicine, Flourtown, PA) 14-year, cost-utility analysis using patient preferences and 2012 United States real dollars.

Visual Impairment and Blindness Avoided with Ranibizumab in Hispanic and Non-Hispanic Whites with Diabetic Macular Edema in the United States.

Objective: To estimate visual impairment (VI) and blindness avoided with intravitreal ranibizumab 0.3 mg treatment for central-involved diabetic macular edema (DME) among Hispanic and non-Hispanic white individuals in the United States.

Design: Population-based model simulating visual acuity (VA) outcomes over 2 years after diagnosis and treatment of DME.

United States comparative costs and absenteeism of diabetic ophthalmic conditions.

Objective: This retrospective cohort study examined the impact of diabetic macular edema (DME), diabetic retinopathy (DR), or diabetes on annual health benefit costs and absenteeism in US employees.

Methods: Claims data from 2001 to 2012 was extracted from the Human Capital Management Services Group Research Reference Database on annual direct/indirect health benefit costs and absences for employees aged ≥ 18 years. Employees with DME, DR, or diabetes were identified by International Classification of Diseases, Ninth Revision, Clinical Modification codes.

Vision-related function after ranibizumab treatment for diabetic macular edema: results from RIDE and RISE.

Objective: To examine the effects of intravitreal ranibizumab (Lucentis; Genentech, Inc., South San Francisco, CA) treatment on patient-reported vision-related function, as assessed by 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores, in patients with visual impairment secondary to center-involved diabetic macular edema (DME).

Design: Within 2 randomized, double-masked, phase 3 clinical trials (RIDE [A Study of Ranibizumab Injection in Subjects With Clinically Significant Macular Edema {ME} With Center Involvement Secondary to Diabetes Mellitus; NCT00473382] and RISE [A Study of Ranibizumab Injection in Subjects With Clinically Significant Macular Edema {ME} With Center Involvement Secondary to Diabetes Mellitus; NCT00473330]), the NEI VFQ-25 was administered at baseline and at the 6-, 12-, 18-, and 24-month follow-up visits.

Prevalence of and risk factors for diabetic macular edema in the United States.

Importance: Diabetic macular edema (DME) is a leading cause of vision loss in persons with diabetes mellitus. Although there are national estimates for the prevalence of diabetic retinopathy and its risk factors among persons with diabetes, to our knowledge, no comparable estimates are available for DME specifically.

Objectives: To estimate the prevalence of DME in the US population and to identify associated risk factors.

Composite Indices Using 3 or 4 Components of the Core Data Set Have Similar Predictive Ability to Measure Disease Activity in RA: Evidence from the DANCER and REFLEX Studies.

Background. Understanding how disease-assessment indices perform in rheumatoid arthritis (RA) clinical trials can inform their use in routine practice. The study objective was to assess the capacity of combinations of RA Core Data Set measures to distinguish rituximab from control treatment.

Underuse of the health care system by persons with diabetes mellitus and diabetic macular edema in the United States.

Importance: Thickening of the center of the retina, diabetic macular edema (DME), is the most common cause of visual loss due to diabetes mellitus. Treatment of DME has improved dramatically, and the prompt diagnosis of DME and referral of these patients have become more critical. Nonetheless, awareness of and care for DME in the US population is uncharacterized.

Patients' willingness to trade off between the duration and frequency of rheumatoid arthritis treatments.

Objective: Biologic treatments for rheumatoid arthritis (RA) vary widely in both the time required to administer treatment and treatment frequency. This study aimed to quantify the rate at which RA patients are willing to trade off between the time required to administer treatment (duration) and treatment frequency.

Methods: Respondents with a self-reported physician diagnosis of moderate to severe RA completed an online discrete choice experiment survey (also known as conjoint analysis).

Retrospective analysis of first-line anti-vascular endothelial growth factor treatment patterns in wet age-related macular degeneration.

Introduction: This study compared the number of, and expenditures on, first-line intravitreal anti-vascular endothelial growth factor (anti-VEGF) injections between patients who were treated with aflibercept or ranibizumab for wet age-related macular degeneration (AMD).

Methods: This was a retrospective cohort study based on U.S.

Risk of infections in rheumatoid arthritis patients switching from anti-TNF agents to rituximab, abatacept, or another anti-TNF agent, a retrospective administrative claims analysis.

Objective: This study compared the incidence and hazard of ICD-9-CM-coded infections and severe infections in rheumatoid arthritis (RA) patients treated with subsequent-line (SL) BIOs (BIO) after switching from first-line (FL) anti-TNF therapy (anti-TNF).

Methods: Retrospective analysis of a large U.S.

How to aggregate health? Separability and the effect of framing.

Background: Unweighted summation or quality-adjusted life year (QALY) utilitarianism is the most common way to aggregate health benefits in a cost-effectiveness analysis. A key qualitative principle underlying QALY utilitarianism is separability: those individuals unaffected by a policy choice should not influence the policy choice. Separability also underlies several of the alternatives for QALY utilitarianism that have been proposed.