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. RESULTS: Using MARINA and HORIZON trial data and a meta-analysis control cohort after 24 months, ranibizumab therapy conferred a combined-eye patient value (quality-of-life) gain of 16.3%, versus 10.4% found in 2006. The two-year direct ophthalmic medical cost for ranibizumab therapy was $46,450, a 33.8% real dollar decrease from 2006. The societal cost perspective CUR was -$242,920/QALY, indicating a $282,517 financial return-on-investment (ROI), or 12.3%/year to society for direct ophthalmic medical costs expended. The 3rd party insurer CUR ranged from $21,199/QALY utilizing all direct, medical costs, to $69,591/QALY using direct ophthalmic medical costs. CONCLUSIONS: Ranibizumab therapy for neovascular AMD in 2015, considering treatment of both eyes, conferred greater patient value gain (comparative effectiveness) and improved cost-effectiveness than in 2006, as well as a large monetary return-on-investment to the Gross Domestic Product and nation's wealth. The model herein integrates important novel features for neovascular age-related macular degeneration, vitreoretinal cost effectiveness analyses, including: (1) treatment of both eyes, (2) a long-term, untreated control cohort, and (3) the use of societal costs.

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. An interviewer-administered, standardized, written survey assessed 1) direct ophthalmic medical, 2) direct nonophthalmic medical, 3) direct nonmedical, and 4) indirect medical costs accrued due solely to neovascular age-related macular degeneration. RESULTS: The mean annual societal cost for the Control Cohort was $6,116 and for the Study Cohort averaged $39,910 (P < 0.001). Study Subcohort 1 costs averaged $20,339, while Subcohort 4 costs averaged $82,984. Direct ophthalmic medical costs comprised 17.9% of Study Cohort societal ophthalmic costs, versus 74.1% of Control Cohort societal ophthalmic costs (P < 0.001) and 10.4% of 20/800 to no light perception subcohort costs. Direct nonmedical costs, primarily caregiver, comprised 67.1% of Study Cohort societal ophthalmic costs, versus 21.3% ($1,302/$6,116) of Control Cohort costs (P < 0.001) and 74.1% of 20/800 to no light perception subcohort costs. CONCLUSION: Total societal ophthalmic costs associated with neovascular age-related macular degeneration dramatically increase as vision in the better-seeing eye decreases.

A Value-Based Medicine cost-utility analysis of genetic testing for neovascular macular degeneration.

BACKGROUND: There is a dearth of patient, preference-based cost-effectiveness analyses evaluating genetic testing for neovascular age-related macular degeneration (NVAMD). METHODS: A Value-Based Medicine, 12-year, combined-eye model, cost-utility analysis evaluated genetic testing of Category 3 AMD patients at age 65 for progression to NVAMD. The benefit of genetic testing was predicated upon the fact that early-treatment ranibizumab therapy (baseline vision 20/40-20/80) for NVAMD confers greater patient value than late-treatment (baseline vision ≤20/160). Published genetic data and MARINA Study ranibizumab therapy data were utilized in the analysis. Patient value (quality-of-life gain) and financial value (2012 US real dollar) outcomes were discounted at 3 % annually. RESULTS: Genetic testing-enabled, early-treatment ranibizumab therapy per patient conferred mean 20/40-1 vision, a 0.845 QALY gain and 14.1 % quality-of-life gain over sham therapy. Late-treatment ranibizumab therapy conferred mean 20/160+2 vision, a 0.250 QALY gain and 4.2 % quality-of-life gain over sham therapy. The gain from early-treatment over late-treatment was 0.595 QALY (10.0 % quality-of-life gain). The per-patient cost for genetic testing/closer monitoring was $2205 per screened person, $2.082 billion for the 944,000 estimated new Category 3 AMD patients annually. Genetic testing/monitoring costs per early-treatment patient totaled $66,180. Costs per early-treatment patient included: genetic testing costs: $66,180 + direct non-ophthalmic medical costs: -$40,914 + caregiver costs: -$172,443 + employment costs: -$14,098 = a net societal cost saving of $160,582 per early treatment patient. When genetic screening facilitated an incremental 12,965 (8.0 %) of the 161,754, new annual NVAMD patients aged ≥65 in the US to undergo early-treatment ranibizumab therapy, each additional patient treated accrued an overall, net financial gain for society of $160,582. Genetic screening was cost-effective, using World Health Organization criteria, when it enabled an incremental 4.1 % (6634) of 161,754 annual NVAMD patients ≥65 years to receive early-treatment ranibizumab therapy. CONCLUSIONS: Genetic screening-enabled, early-treatment ranibizumab therapy for NVAMD is cost-effective if it enables an incremental 4.1 % of the annual US cohort of new-onset NVAMD patients ≥65 to undergo early-treatment with ranibizumab.

Financial return-on-investment of ophthalmic interventions: a new paradigm.

PURPOSE OF REVIEW: Although the patient value gain (improvement in quality-of-life and/or length-of-life) has been highlighted in Value-based Medicine cost-utility analyses, the financial value gain associated with healthcare interventions has received less emphasis. It is important for professional healthcare providers to realize their interventions often confer a large financial return-on-investment (ROI) to society. RECENT FINDINGS: The societal costs associated with vitreoretinal and other ophthalmic interventions include: direct ophthalmic medical costs expended (hospital, physician, drug, diagnostic testing and so forth), direct medical costs saved (decreased costs for depression, injury, skilled nursing facility, nursing home and others), direct nonmedical costs saved (decreased costs for caregivers, transportation, residence costs, moving costs, and others), and indirect medical costs saved (improving employment incidence and wages). The financial ROI for direct ophthalmic medical costs expended for ranibizumab therapy for neovascular age-related macular degeneration is 450%, whereas that for cataract surgery is 4500% and for medical open-angle glaucoma therapy is 4000%. Many costs gained add to the Gross Domestic Product and increase the wealth of the nation. SUMMARY: Many vitreoretinal and other ophthalmologic interventions confer considerable patient value, but also result in a large financial ROI to society. This financial ROI increases the wealth of the nation.

Cataract surgery cost utility revisited in 2012: a new economic paradigm.

OBJECTIVE: To assess the 2012 cost utility of cataract surgery in the United States and to compare 2012 cost-utility data with those from 2000. DESIGN: Value-Based Medicine (Flourtown, PA), patient preference-based, comparative effectiveness analysis and cost-utility analysis using 2012 real United States dollars. PARTICIPANTS: Previously published Patient Outcomes Research Team Study data and time tradeoff utilities obtained from patients with vision loss. Visual acuity measurements from patients wtih untreated cataract were used as controls. INTERVENTION: Thirteen-year, average, first-eye and second-eye cataract surgery cost-utility analysis using the societal and third-party insurer cost perspectives. MAIN OUTCOME MEASURES: Patient value gain in quality-adjusted life years (QALYs) and percent gain in quality of life as well as the cost-utility ratio using the dollars expended per QALY gained. Patient and financial value outcomes were discounted at 3% annually with net present value analysis. RESULTS: First-eye cataract surgery conferred 1.6212 QALYs over the 13-year model, a 20.8% quality-of-life gain. Bilateral cataract surgery conferred 2.8152 QALYs over 13 years, a 36.2% improvement in quality of life. The direct ophthalmic medical cost for unilateral cataract surgery in 2012 United States nominal dollars was $2653, an inflation-adjusted 34.2% less than in 2000 and 85% less than in 1985. The 2012 inflation-adjusted physician fee was 10.1% of that in 1985. The 13-year societal cost perspective, financial return on investment (ROI) for first-eye cataract surgery was $121,198, a 4567% gain. The third-party insurer cost perspective average cost-utility ratio was $2653/1.6212 = $1636/QALY for unilateral cataract surgery, whereas the societal cost perspective average cost-utility ratio was -$121,198/1.6212 = -$74,759/QALY. The net 13-year $123.4-billion financial ROI from a 1-year cohort of cataract surgery patients was accrued: Medicare, $36.4 billion; Medicaid, $3.3 billion; other insurers, $9.6 billion; patients, $48.6 billion; and increased United States national productivity, $25.4 billion. CONCLUSIONS: Cataract surgery in 2012 greatly improved quality of life and was highly cost effective. It was 34.4% less expensive than in 2000 and 85% less expensive than in 1985. Initial cataract surgery yielded an extraordinary 4567% financial ROI to society over the 13-year model.

Preference-based comparative effectiveness and cost­effectiveness: a review and relevance of value-based medicine for vitreoretinal interventions.

PURPOSE OF REVIEW: This analysis discusses the comparative effectiveness and cost-effectiveness of vitreoretinal interventions, measured in quality-adjusted life years (QALYs) and percentage patient value (PPV gain, or improvement in quality of life and/or length of life). The material is relevant since the Patient Protection and Affordable Care Act enacted by Congress with the support of the President has emphasized the critical importance of patient-based preferences. RECENT FINDINGS: The majority of preference-based, comparative effectiveness and cost-effectiveness vitreoretinal interventions assessed in the US healthcare literature are Value-Based Medicine analyses, thus comparable. These interventions confer a mean patient (human) value gain (improvement in quality of life) of 8.3% [SD 6.3%, 95% confidence interval (CI) + 2.6%]. The average cost-utility of these vitreoretinal interventions is US$23 026/QALY (SD US$24 508, 95% CI + US$8770). Most vitreoretinal interventions are very cost effective using a conventional US standard of US$50 000/QALY as the upper anchor for a very cost-effective intervention, and the World Health Organization of approximately US$142 200/QALY as the upper anchor for a cost-effective intervention. CONCLUSIONS: Most vitreoretinal interventions confer considerable patient value and are very cost effective. Further standardization across healthcare is needed in the preference-based, comparative and cost-utility (cost-effectiveness) arena. The metrics of PPV (percentage patient value) gain and US$/PPV (dollars expended per percentage patient value gain) or financial value gain may be more user-friendly than the QALY.

Comparative effectiveness and cost-effectiveness of the implantable miniature telescope.

OBJECTIVE: To assess the preference-based comparative effectiveness (human value gain) and the cost-utility (cost-effectiveness) of a telescope prosthesis (implantable miniature telescope) for the treatment of end-stage, age-related macular degeneration (AMD). DESIGN: A value-based medicine, second-eye model, cost-utility analysis was performed to quantify the comparative effectiveness and cost-effectiveness of therapy with the telescope prosthesis. PARTICIPANTS: Published, evidence-based data from the IMT002 Study Group clinical trial. Ophthalmic utilities were obtained from a validated cohort of >1000 patients with ocular diseases. METHODS: Comparative effectiveness data were converted from visual acuity to utility (value-based) format. The incremental costs (Medicare) of therapy versus no therapy were integrated with the value gain conferred by the telescope prosthesis to assess its average cost-utility. The incremental value gains and incremental costs of therapy referent to (1) a fellow eye cohort and (2) a fellow eye cohort of those who underwent intra-study cataract surgery were integrated in incremental cost-utility analyses. All value outcomes and costs were discounted at a 3% annual rate, as per the Panel on Cost-Effectiveness in Health and Medicine. MAIN OUTCOME MEASURES: Comparative effectiveness was quantified using the (1) quality-adjusted life-year (QALY) gain and (2) percent human value gain (improvement in quality of life). The QALY gain was integrated with incremental costs into the cost-utility ratio ($/QALY, or US dollars expended per QALY gained). RESULTS: The mean, discounted QALY gain associated with use of the telescope prosthesis over 12 years was 0.7577. When the QALY loss of 0.0004 attributable to the adverse events was factored into the model, the final QALY gain was 0.7573. This resulted in a 12.5% quality of life gain for the average patient during the 12 years of the model. The average cost-utility versus no therapy for use of the telescope prosthesis was $14389/QALY. The incremental cost-utility referent to control fellow eyes was $14063/QALY, whereas the incremental cost-utility referent to fellow eyes that underwent intra-study cataract surgery was $11805/QALY. CONCLUSIONS: Therapy with the telescope prosthesis considerably improves quality of life and at the same time is cost-effective by conventional standards.