Evaluating the Relationship Between Visual Acuity and Utilities in Patients With Diabetic Macular Edema Enrolled in Intravitreal Aflibercept Studies.

Purpose: The purpose of this study was to explore the relationship between visual acuity and utility (health-related quality of life) in diabetic macular edema (DME) using intravitreal aflibercept data. Methods: The relationship between visual acuity in the best-seeing eye (BSE) and worse-seeing eye (WSE) and utility was explored using ordinary least squares (OLS) and random-effects models adjusted for different covariates (age, age2, sex, body mass index, smoking status, glycated hemoglobin, diabetes severity, comorbidities, and geographic region). Utility was measured using the EuroQoL-five dimensions questionnaire (EQ-5D) and Visual Functioning Questionnaire-Utility Index (VFQ-UI). For each model, coefficients (R2) were reported, and WSE/BSE was expressed as the ratio of coefficients (OLS models). Models were independent of treatment effects, and outcomes from all time points (up to week 100) were included where available. Results: Data from 1320 patients with DME were analyzed. In all models, the association between visual acuity (BSE > WSE) was stronger with VFQ-UI- than EQ-5D-derived utilities. The estimated relationship between VFQ-UI and visual acuity in the BSE and WSE was robust, even with an increasing number of covariates. WSE/BSE coefficient ratios were similar across VFQ-UI OLS models (32%) compared with EQ-5D models (41%-48%). Actual (unadjusted) versus predicted data plots also showed a better fit with VFQ-UI- than EQ-5D-derived utilities. Conclusions: These analyses show that VFQ-UI was more sensitive than EQ-5D-derived utilities for measuring the impact of visual acuity in the BSE and WSE. Visual acuity in the BSE was a major contributor to utility, but WSE is also important though to a lesser degree as shown by the coefficient ratios. These new data will be useful for health technology assessments in DME, where utilities data are lacking.

Cost-effectiveness of intravitreal aflibercept versus other treatments for wet age-related macular degeneration in Japan.

OBJECTIVE: This analysis estimated the cost-effectiveness of intravitreal aflibercept injection(s) (IAI) for wet age-related macular degeneration (wAMD) compared with other treatments in Japan. METHODS: This was a cost-utility analysis based on published data. A state-transition cohort model was constructed with six health states based on best-corrected visual acuity in the better-seeing eye. The cycle time was 4 weeks, and the time horizon was 12 years. The model compared IAI 2 mg every 8 weeks (2q8) for 2 years after three initial monthly injections, ranibizumab as needed, ranibizumab 0.5 mg every 4 weeks (0.5q4), pegaptanib sodium 0.3 mg every 6 weeks, verteporfin photodynamic therapy (PDT), and best supportive care, assumed to include medical management and monitoring, but no active therapy. Costs (expressed as Japanese yen [JPY]) and quality-adjusted life years (QALYs) gained were estimated for each treatment and discounted at 2.0%. Input data were obtained from clinical studies, the Japanese drug tariff and social insurance reimbursement schedule, and expert opinion. The analysis was conducted from the societal perspective, including medical costs as well as costs of blindness. RESULTS: IAI 2q8 was dominant (i.e. more effective in terms of QALYs and less costly) to all other comparators (ranibizumab as needed, ranibizumab 0.5q4, pegaptanib sodium, PDT, and best supportive care), as shown by the incremental cost-utility ratio (i.e. cost per QALY gained). LIMITATIONS: The strengths of the analysis include the wide range of comparators evaluated and the use of Japanese-specific utility data. The limitations include the use of one eye, inclusion of published data up to 2 years only, and assumptions on disease course over 5 years. CONCLUSIONS: IAI 2q8 was more effective in terms of QALYs and less costly compared with other treatments for wAMD in Japan.

Ipilimumab in 2nd line treatment of patients with advanced melanoma: a cost-effectiveness analysis.

OBJECTIVE: To estimate the cost-effectiveness of ipilimumab (3 mg/kg) compared with best supportive care (BSC) in pre-treated advanced melanoma patients. METHODS: The analysis was based on a US payer perspective and lifetime time horizon. A three-state Markov model was developed representing clinical outcomes, quality-of-life, and healthcare resource use of patients treated with ipilimumab and BSC. Transitions between states were modeled using overall and progression-free survival data from the MDX010-20 trial. Utility data were from a melanoma-specific study of the health state preferences of the general population. Disease management costs expressed in 2011 US Dollars were based on healthcare resource use observed in a US retrospective medical chart study. Uncertainty was analyzed using one-way and probabilistic sensitivity analyses. RESULTS: The gain in life years and QALYs from introducing ipilimumab over BSC were 1.88 years (95% CI = 1.62-2.20) and 1.14 (95% CI = 1.01-1.34) QALYs, respectively, over the lifetime time horizon. The estimated incremental cost of treating with ipilimumab vs BSC was $146,716 (95% CI = $130,992-$164,025). The estimated incremental cost-effectiveness ratios were $78,218 per life year gained and $128,656 per QALY gained. Ipilimumab was 95% likely to be cost-effective at a willingness-to-pay of $146,000/QALY. LIMITATIONS: Ipilimumab's method of action causes a tumor response pattern that differs from the Response Evaluation Criteria in Solid Tumors upon which the model is based, leading to a potential under-estimate of quality-of-life of ipilimumab patients. Survival and QALY gains were related to the time horizon of the analysis. Sensitivity analyses indicated that qualitative conclusions regarding the cost-effectiveness of ipilimumab were unchanged when the method of quality adjustment and the time horizon were varied. CONCLUSION: The analysis shows that the estimated cost-effectiveness of ipilimumab is within what has been shown to be acceptable to payers for oncology products in the US.