Cost-effectiveness of voretigene neparvovec in the treatment of patients with inherited retinal disease with RPE65 mutation in Switzerland.

OBJECTIVE: We aimed to evaluate the cost-effectiveness of voretigene neparvovec (VN) compared with standard of care (SoC) for patients with inherited retinal disease (IRD) caused by a biallelic RPE65-mutation. VN is a live, non-replicating adeno-associated virus serotype 2 (AAV2). SoC is best supportive care provided to patients with visual impairment. Patients under SoC may experience progressive vision loss leading to complete blindness. METHODS: We adapted a previously published Markov cohort model for IRD. An annual cycle length, life-long time horizon, discount rate of 3% for cost and health outcomes, and Swiss health system perspective were used. Data from a randomised controlled phase III trial of VN versus SoC (ClinicalTrials.gov: NCT00999609) were used to estimate transitions between health states in the first year, after which VN patients were assumed to remain for 39 subsequent years in the health state they were in at the end of the first year. After the 40th year for VN patients and 1st year for SoC patients, visual decline was modelled based on observational data on the natural progression of the disease. Quality-adjusted life years (QALYs) were calculated based on an external study which elicited clinicians' EQ-5D-5L-based utility estimates for IRD patients with a RPE65-mutation. Costs (Swiss Francs (CHF), year 2018-2019) included drug acquisition/ administration, adverse events, testing for sufficient viable retinal cells, and healthcare-related costs of blindness. Societal costs of blindness were added in a complementary analysis. Robustness of the model results were tested in sensitivity and scenario analyses. RESULTS: For the base-case, VN resulted in incremental costs per patient of CHF 764'402 (VN: CHF 901'654, SoC: CHF 137'252), incremental blindness-free years of 7.67 (VN: 28.32, SoC: 20.65) and incremental QALYs of 6.73 (VN: 18.35, SoC: 11.62), leading to an incremental cost-effectiveness ratio of CHF 113'526 per QALY gained. In probabilistic sensitivity analysis, the cost-effectiveness of VN was better than CHF 100,000 per QALY gained in 41% of iterations. For the scenario analysis in which a societal perspective was adopted and for which a 50% work-related productivity loss from blindness was assumed, incremental costs of CHF 423,837 and an ICER of CHF 62'947 per QALY gained were produced. The scenario assuming VN treatment effect lasts for 20 years produced an ICER of CHF 156'171 per QALY gained, whereas assuming a life-long VN treatment effect resulted in an ICER of CHF 96'384 per QALY gained. CONCLUSION: The incremental cost-effectiveness ratio of VN compared to the SoC was estimated to be CHF 113'526 and CHF 62'947 per QALY gained, respectively, from a Swiss healthcare system, and societal perspective assuming a 50% productivity loss.

TRENDS IN INTRAVITREAL CORTICOSTEROID AGENT USE BY US OPHTHALMOLOGISTS IN MEDICARE BENEFICIARIES AND ASSOCIATION WITH PHYSICIAN-INDUSTRY INTERACTIONS.

PURPOSE: To report trends of intravitreal corticosteroid use and explore the relationship between career experience, reported industry payments, and prescribing habits. METHODS: A retrospective review of ophthalmologists who administered intravitreal dexamethasone implants (DEX) and triamcinolone acetonide (TA) injections between August 2013 and December 2017. RESULTS: A total of 1,070 US ophthalmologists were reimbursed by Medicare for 522,804 DEX injections and 2.6 million TA injections. There was a significant positive trend in the number of DEX (P = 0.01), but not TA, injections per year. Mid-career and late-career physicians performed significantly greater total injections on average compared with early-career physicians (both P < 0.001). Early-career physicians performed a greater proportion of DEX injections than late-career physicians (P = 0.006). Industry payments were positively associated with the proportion of DEX used and inversely correlated with the proportion of TA administered (P < 0.001). On multivariate analysis, years in practice, number of payments, and total value of payments were significantly associated with the number of DEX injections administered (all P < 0.001). CONCLUSION: From 2013 to 2017, the use of DEX increased, whereas TA use remained stable. There was a positive association between DEX use and physician-industry interactions, which may be explained by seniority and experience. This study does not define a causal relationship.

Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease.

OBJECTIVE: The gene therapy voretigene neparvovec (VN) is the first Food and Drug Administration-approved treatment for vision loss owing to the ultra-rare RPE65-mediated inherited retinal disorders. We modeled the cost-utility of VN compared with standard of care (SoC). STUDY DESIGN: A 2-state Markov model, alive and dead, with a lifetime horizon. METHODS: Visual acuity (VA) and visual field (VF) were tracked to model quality-adjusted life-years (QALYs). VN led to an improvement in VA and VF that we assumed was maintained for 10 years followed by a 10-year waning period. The cost of VN was $850 000, and other direct medical costs for depression and trauma were included for a US healthcare system perspective. A modified societal perspective also included direct nonmedical costs and indirect costs. RESULTS: VN provided an additional 1.3 QALYs over the remaining lifetime of an individual. The average total lifetime direct medical cost for individuals treated with VN was $1 039 000 compared with $213 400 for SoC, leading to an incremental cost-effectiveness ratio (ICER) of $643 800/QALY from the US healthcare system perspective. Direct nonmedical costs totalled $1 070 900 for VN and $1 203 300 for SoC, and indirect costs totalled $405 400 for VN and $482 900 for SoC, leading to an ICER of $480 100/QALY from the modified societal perspective. CONCLUSIONS: At the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC. VN has important implications for both development and pricing of future gene therapies; therefore clinical and economic analyses must be carefully considered.

Costs and benefits of bevacizumab vial sharing for the treatment of retinal diseases.

BACKGROUND: Antiangiogenic therapy has proved to be an important therapeutic tool for many retinal vascular diseases; however, its availability is limited in developing countries. This study sought to describe the bevacizumab vial sharing process and to evaluate the impact of this repackaging system on the costs incurred in a Brazilian public hospital. METHOD: This retrospective study compared the number and costs of intravitreal antiangiogenic injections approved via court order in the first year of the study (2015) to the number and costs of the bevacizumab injections provided through the use of vial sharing in the second year of the study (2016). Vial sharing consists of the traditional process used to repackage bevacizumab; in this case, however, the drug samples used were the residual volume from the preparation of bevacizumab for oncology patients. The hospital adhered to the guidelines established by the Brazilian Health Surveillance Agency (ANVISA). RESULTS: In the first year of the study and using medication obtained through court orders, 550 intravitreal injections were performed in the ophthalmology ambulatory care center. Based on local pricing tables, the total cost of the medication was BRL$1,036,056.25 (USD$267,546.58), and the average cost of each application was BRL$1883.74 (USD$486.45). In the second year of the study, 1081 intravitreal applications were performed at the same hospital using doses obtained through bevacizumab vial sharing. The total cost was BRL$21,942.49 (USD$5663.30) and the per-unit cost was BRL$20.30, or USD$5.23 (a savings of 97.88%). CONCLUSION: This study found that bevacizumab vial sharing led to a significant reduction in public health care costs associated with antiangiogenic treatment and increased the availability of the drug to public health care patients. These results can be extrapolated to other types of drugs and health care systems.

Outcomes and costs of Ranibizumab and Aflibercept treatment in a health-service research context.

BACKGROUND: To compare anti-VEGF treatments for macular disease in terms of costs and clinical outcomes. METHODS: We identified patients suffering from macular disease and treated either with aflibercept, ranibizumab or both at the largest public eye clinic in Switzerland between January 1st and December 31st 2016 who were insured in one of the two participating health insurance companies. Clinical data were extracted from the electronic health record system. The health insurers provided the health claim costs for the ophthalmologic care and the total health care costs of each patient in the observation period. Using multivariate regression models, we assessed the monthly ophthalmologic and the monthly total costs of patients with no history of switching (ranibizumab vs. aflibercept), patients with a history of switching from ranibizumab to aflibercept, patients switching during the observation period and a miscellaneous group. We examined baseline differences in age, proportion of males, visual acuity (letters), central retinal thickness (CRT) and treatment history before entering the study. We investigated treatment intensity and compared the changes in letters and CRT. RESULTS: The analysis involved 488 eyes (361 patients), 182 on ranibizumab treatment, and 63 on aflibercept treatment, 160 eyes with a history of switching from ranibizumab to aflibercept, and 45 switchers during follow-up and 38 eyes of the miscellaneous group. Compared to ranibizumab, monthly costs of ophthalmologic treatment were slightly higher for aflibercept treatment + 175.0 CHF (95%CI: 1.5 CHF to 348.3 CHF; p = 0.048) as were the total monthly costs + 581.0 CHF (95%CI: 159.5 CHF to 1002.4 CHF; p = 0.007). Compared to ranibizumab, the monthly treatment intensity with aflibercept was similar (+ 0.057 injections/month (95%CI -0.023 to 0.137; p = 0.162), corresponding to a projected annual number of 5.4 injections for ranibizumab vs. 6.1 injections for aflibercept. During follow-up, visus dropped by 0.7 letters with ranibizumab and increased by 0.6 letters with aflibercept (p = 0.243). CRT dropped by - 14.9 µm with ranibizumab and by - 19.5 µm with aflibercept (p = 0.708). The monthly costs of all other groups examined were higher. CONCLUSION: These real-life data show that aflibercept treatment is equally expensive, and clinical outcomes between the two drugs are similar.

AIMING FOR THE BULL'S EYE: The Cost-Utility of Screening for Hydroxychloroquine Retinopathy.

BACKGROUND: Throughout medicine, the cost of various treatments has been increasingly studied with the result that certain management guidelines might be reevaluated in their context. Cost-utility is a term referring to the expense of preventing the loss of quality of life, quantified in dollars per quality-adjusted life year. In 2002, the American Academy of Ophthalmology published hydroxychloroquine screening recommendations which were revised in 2011. The purpose of this report is to estimate the cost-utility of these recommendations. METHODS: A hypothetical care model of screening for hydroxychloroquine retinopathy was formulated. The costs of screening components were calculated using 2016 Medicare fee schedules from the Centers for Medicare and Medicaid Services. RESULTS: The cost-utility of screening for hydroxychloroquine retinopathy with the 2011 American Academy of Ophthalmology guidelines was found to vary from 33,155 to 344,172 dollars per quality-adjusted life year depending on the type and number of objective screening tests chosen, practice setting, and the duration of hydroxychloroquine use. Screening had a more favorable cost-utility when the more sensitive and specific diagnostics were used, and for patients with an increased risk of toxicity. CONCLUSION: American Academy of Ophthalmology guidelines have a wide-ranging cost-utility. Prudent clinical judgment of risk stratification and tests chosen is necessary to optimize cost-utility without compromising the efficacy of screening.

Cost evaluation of surgical and pharmaceutical options in treatment for vitreomacular adhesions and macular holes.

OBJECTIVE: To evaluate cost-effectiveness and cost utilities for treatment options for vitreomacular adhesions (VMAs) and full-thickness macular holes (MHs). DESIGN: A Markov model of cost-effectiveness and utility. PARTICIPANTS: There were no participants. METHODS: Outcomes of published clinical trials (index studies) of surgical treatment of VMAs and MHs and a prospective, multicenter clinical trial of pharmaceutical vitreolysis with intravitreal ocriplasmin with saline control were used to generate a model for costs of treatment and visual benefits. All techniques were assumed to result in a 2.5-line visual benefit if anatomy was resolved. Markov analysis, with cost data from the Centers for Medicare and Medicaid Services, was used to calculate imputed costs for each primary treatment modality in a facility setting, with surgery performed in a hospital serving as the highest end of the range and nonfacility setting with surgery performed in an ambulatory surgery center serving as the lowest end of the range. MAIN OUTCOME MEASURES: Imputed costs of therapy, cost per line saved, cost per line-year saved, cost per quality-adjusted life years (QALYs). RESULTS: When pars plana vitrectomy (PPV) was selected as the primary procedure, the overall imputed cost ranged from $5802 to $7931. The cost per line was $2368 to $3237, the cost per line-year saved was $163 to $233 and the cost per QALY was $5444 to $7442. If intravitreal injection of ocriplasmin was the primary procedure, the overall imputed cost was $8767 to $10 977. The cost per line ranged from $3549 to $4456, the cost per line-year saved was $245 to $307, and the cost per QALY was between $8159 and $10 244. If intravitreal saline injection was used as a primary procedure, the overall imputed cost was $5828 to $8098. The cost per line was $2374 to $3299, the cost per line-year saved was $164 to $227, and the cost per QALY was $5458 to $7583. CONCLUSIONS: As a primary procedure, PPV was the most cost-effective therapy in this model. The other treatments had similar costs per QALY saved and compare favorably with costs of therapy for other retinal diseases.

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.

Cost Analysis of Routine Vitrectomy Surgery.

PURPOSE: To use electronic health record (EHR) time logs to calculate the complete cost profiles of routine pars plana vitrectomy surgery. DESIGN: Economic analysis. PARTICIPANTS: Patients undergoing elective vitrectomy procedures (Current Procedural Terminology codes 67040, 67041, and 67042) at Vanderbilt University Medical Center in fiscal year 2019. METHODS: Process flow mapping for routine vitrectomy surgery was used to define the operative episode. De-identified time logs were sourced from an internal perioperative data warehouse to calculate procedure-level durations. The costs of materials and overhead were calculated from internal financial management software. Costs per minute for space, equipment, and personnel were based on internal figures. These inputs were used for a time-driven activity-based costing (TDABC) analysis. MAIN OUTCOME MEASURES: Complete cost profile of routine pars plana vitrectomy surgery. RESULTS: Cost analysis of routine vitrectomy surgery resulted in a total cost of $7169.79 per patient, which was $2053.85 more than the maximum Medicare reimbursement for the equivalent episode, $5115.93. Vitrectomy cases do not break even unless the case duration is fewer than 26.81 minutes, overhead is reduced by 53.78%, or reimbursement is increased by 40.15%. Reimbursement does not compensate for variable costs alone for cases lasting longer than 55.09 minutes. In the cohort used here, 68% of cases are completely unprofitable, with increasing losses directly proportional to the length of the case. CONCLUSIONS: This analysis showed that true costs for routine vitrectomy procedures are significantly more than the maximum allowable Medicare reimbursement. Academic ophthalmology departments may benefit from more accurate costing approaches using existing EHR data. These approaches may be informative for policy discussion regarding appropriate reimbursement.

A Multinational Comparison of Anti-Vascular Endothelial Growth Factor Use: The United States, the United Kingdom, and Asia-Pacific.

PURPOSE: A comparison of anti-vascular endothelial growth factor (anti-VEGF) medication use across multiple countries. CLINICAL RELEVANCE: Anti-VEGF medication use is now considered first-line treatment for numerous retinal diseases globally. Exploring medication choices, costs within each healthcare system, policy challenges, emerging treatments, and patient access all provide insight into a newly recognized and major public health issue. METHODS: All data presented in this review are available through the published English literature in PubMed, non-peer-reviewed trade publications, and reported surveys. The following search terms were used: anti-VEGF OR bevacizumab OR ranibizumab OR aflibercept OR pegaptanib OR conbercept AND trends OR survey OR cost OR patterns OR preference. Countries with large populations and available data included the United States, United Kingdom, China, India, Korea, Singapore, and Australia. Population and economic statistics were obtained from published reports from the World Bank, World Health Organization, and Commonwealth Fund. RESULTS: Anti-VEGF medication use and costs are significant aspects of patient and healthcare system expenditures in each nation and may have an especially large potential economic burden in India and China. Bevacizumab use comprises the majority of anti-VEGF medication use in the United States and Singapore, although aflibercept use is growing rapidly. Paradoxically, data demonstrate that there is a significant trend in medication choice toward ranibizumab and aflibercept among practice settings outside of the United States, such as the United Kingdom, China, South Korea, and Australia. The price of anti-VEGF medications ranged from US $30 (ziv-aflibercept) to US $1950 (ranibizumab and aflibercept). Ranibizumab's price ranged from US $240 in India to US $1950 in the United States. Conbercept in China costs approximately US $1150 per dose. CONCLUSIONS: Outside of the United States, many nations are using a majority of more expensive anti-VEGF medications, which may lead to increased costs and decreased access. Increasing the availability of safely compounded anti-VEGF medications will likely improve access, create patient/provider choice, and decrease relative healthcare costs for the growing burden of retinal diseases globally.

Economic Evaluation of Low-Vision Rehabilitation for Veterans With Macular Diseases in the US Department of Veterans Affairs.

Importance: Examining costs and consequences of different low-vision (LV) programs provides important information about resources needed to expand treatment options efficiently. Objective: To examine the costs and consequences of LV rehabilitation or basic LV services. Design, Setting, and Participants: The US Department of Veterans Affairs (VA) Low Vision Intervention Trial (LOVIT) II was conducted from September 27, 2010, to July 31, 2014, at 9 VA facilities and included 323 veterans with macular diseases and a best-corrected distance visual acuity of 20/50 to 20/200. Veterans were randomized to receive basic LV services that provided LV devices without therapy, or LV rehabilitation that added a therapist to LV services who provided instruction and homework on using LV devices, eccentric viewing, and environmental modification. We compared costs and consequences between these groups. Interventions: Low-vision devices without therapy and LV devices with therapy. Main Outcomes and Measures: Costs of providing basic LV services or LV rehabilitation were assessed. We measured consequences as changes in functional visual ability from baseline to follow-up 4 months after randomization using the VA Low Vision Visual Functioning Questionnaire. Visual ability was measured in dimensionless log odds units (logits). Results: Of 323 randomized patients, the mean (SD) age was 80 (10.5) years, 314 (97.2%) were men, and 292 (90.4%) were white. One hundred sixty (49.5%) received basic LV services and 163 (50.1%) received LV rehabilitation. The mean (SD) total direct health care costs per patient were similar between patients who were randomized to receive basic LV services ($1662 [$671]) or LV rehabilitation ($1788 [$864]) (basic LV services, $126 lower; 95% CI, $299 lower to $35 higher; P = .15). However, basic LV services required less time and had lower transportation costs. Patients receiving LV rehabilitation had greater improvements in overall visual ability, reading ability, visual information processing, and visual motor skill scores.

[Cost recovery for the treatment of retinal and vitreal diseases by pars plana vitrectomy under the German DRG system]. / Kosteneffizienz von netzhaut- und glaskörperchirurgischen Eingriffen mittels ppV unter DRG-Bedingungen.

BACKGROUND: Since 2004 inpatient health care in Germany is paid according to calculated DRGs. Only a few university hospitals participated in distinct cost calculations of clinical treatment. It was the aim of this study to check the cost recovery at a University Eye Hospital for the surgical treatment of retinal and vitreal diseases by pars plana vitrectomy (ppV), which are included in DRGs C03Z and C17Z. MATERIAL AND METHODS: The performance data for both DRGs were collected for the years 2005 and 2006 using the E1 sheets according to section 21 KHEntG. The mean duration of all procedures was collected by data from the internal controlling. Costs for single operations were calculated from fixed and variable costs for the operation theatre and the ward including costs for personnel and material. RESULTS: In the 2-year period of 4,721 inpatient procedures 1,307 ppVs were performed. Each ppV had fixed surgical costs of 130.60 EUR; personnel costs varied between 575 EUR (C03Z; including cataract surgery; mean OP duration: 85 min) and 510 EUR (C17Z; no cataract surgery; mean OP duration: 73 min) at a proportion between general anaesthesia and local anaesthesia of 80/20. For a pure ppV material costs were 255 EUR. Additional adjuncts such as an encircling band, perfluorcarbon, ICG, tPA, gas and silicon oil or cataract surgery led to extra costs between 51 EUR and 250 EUR per adjunct und were used in 56% (C03Z) and 74.5% (C17Z) of all procedures. Costs for hospitalisation were about 1765 EUR at a mean residence time of 6.5 days. Thus, the overall costs of a pure basic ppV amounted to 2975 EUR (C03Z) and 2661 EUR (C17Z). In consideration of the current relative DRG weights of 1.08 and 0.957 and a current base rate of 2787.19 EUR in Bavaria, cost recovery is only given for basic ppV but not for complex ppVs having higher material and personnel costs. Additionally, the costs for multiple surgeries as occur in 5.9% of cases are not compensated by the DRG system. CONCLUSION: The reimbursement for inpatient ppVs in a University environment is not covered for complex procedures requiring more cost-effective material and personnel time. To consider an adequate cost recovery for these procedures a DRG split for both DRGs (C03Z and C17Z) in basic ppVs and complex ppVs is required. We recommend this proposal for the InEK.

Intravitreal Ziv-Aflibercept: Clinical Effects and Economic Impact.

During the past decade, drugs that inhibit the actions of vascular endothelial growth factor (VEGF) have become standard-of-care treatment for a variety of chorioretinal vascular conditions. The off-label, intravitreal use of ziv-aflibercept (Zaltrap) has provided clinicians with an additional cost-effective drug. The commercial preparation of ziv-aflibercept contains the same aflibercept (VEGF-trap) molecule as Eylea but has a much higher osmolarity (1000 mOsm/kg vs 300 mOsm/kg). Initial concerns regarding cytotoxicity and long-term safety of intravitreal ziv-aflibercept have been largely negated after a series of publications failed to identify adverse ocular and systemic side effects. Both treatment-naive and anti-VEGF‒resistant cases of neovascular age-related macular degeneration (nAMD), diabetic macular edema (DME), retinal vein occlusion (RVO), and choroidal neovascular membrane (CNVM) may respond as well to ziv-aflibercept as to aflibercept. A higher dose of ziv-aflibercept (2 mg in 0.08 mL) does not cause any adverse effects during short-term follow-up period (1 month). Data from various sources suggest that ziv-aflibercept may be as cost effective as bevacizumab, thereby making it an attractive treatment option in low- and middle-income countries. However, problems with off-label use, compounding, and counterfeiting limit its availability in many countries. Data from prospective, randomized, multicenter clinical trials are still required to convince physicians and regulatory bodies of its clinical efficacy and potential as early therapy.

Regulatory and Economic Considerations of Retinal Drugs.

The advent of anti-VEGF therapy for neovascular age-related macular degeneration and macular edema secondary to retinal vein occlusion and diabetes mellitus has prevented blindness in tens of thousands of people. However, the costs of these drugs are without precedent in ophthalmic drug therapeutics. An analysis of the financial implications of retinal drugs and the impact of the Food and Drug Administration on treatment of retinal disease must include not only an evaluation of the direct costs of the drugs and the costs associated with their administration, but also the cost savings which accrue from their clinical benefit. This chapter will discuss the financial and regulatory issues associated with retinal drugs.

Review of economic studies and budget impact analysis of ocriplasmin as a treatment of vitreomacular traction. / Revisión de estudios económicos y análisis de impacto presupuestario de la ocriplasmina como tratamiento de la tracción vitreomacular.

OBJECTIVE: To review the evidence on the cost-effectiveness of ocriplasmin as a treatment for vitreomacular traction (VMT), and to estimate the impact on the Spanish National Health System (NHS). MATERIAL AND METHODS: 1) Systematic review. The following databases were searched in January 2015: MEDLINE, PREMEDLINE, EMBASE, CRD, the Cochrane Library, and key websites. Selection criteria were: full economic evaluations that compared ocriplasmin with usual care ('watch and wait' and/or vitrectomy) in patients with VMT. The outcomes to extract were costs of the alternatives and the incremental cost-effectiveness ratio. Studies of budget impact analysis were also included. The methodological quality was assessed, and a narrative synthesis of the included studies was carried out. 2) Estimation of budget impact. The impact on the budget as a result of the introduction of ocriplasmin in the NHS was estimated, including data from different sources. RESULTS: Six studies were identified, none of them performed in Spain. The two best studies concluded that ocriplasmin is cost-effective in their respective countries (Canada and United Kingdom), but only in patients with certain conditions (without epiretinal membrane, for example). The results of the budget impact analysis are different between countries. The analysis for Spain showed that the introduction of ocriplasmin would mean a saving over 1 million Euros for the NHS in 5 years. CONCLUSIONS: The cost-effectiveness of ocriplasmin has not been demonstrated in Spain. However, good studies performed in other countries found that ocriplasmin is cost-effective in selected patients. Given the current prices in Spain, ocriplasmin could involve a saving for the Spanish NHS.

What Is Next for Retinal Gene Therapy?

The field of gene therapy for retinal blinding disorders is experiencing incredible momentum, justified by hopeful results in early stage clinical trials for inherited retinal degenerations. The premise of the use of the gene as a drug has come a long way, and may have found its niche in the treatment of retinal disease. Indeed, with only limited treatment options available for retinal indications, gene therapy has been proven feasible, safe, and effective and may lead to durable effects following a single injection. Here, we aim at putting into context the promise and potential, the technical, clinical, and economic boundaries limiting its application and development, and speculate on a future in which gene therapy is an integral component of ophthalmic clinical care.

The value of routine dilated pupil screening examination.

We evaluated the cost-effectiveness of routine dilated fundus examination in improving visual outcomes. The cost of routine dilated fundus examination was related to the number of preventable cases of vision-threatening peripheral retinal disease. Patients with these diseases who had no risk factors were ascertained in a population of 1.75 million adults for a period of 6 months. Those whose last examination had been undilated were identified because only for them could routine dilated examination (RDE) have been substituted for undilated examination. The number of preventable cases was calculated for multiples of a 10% probability of prevention. The cost of RDE was determined from the number of undilated examinations in the same population and period and the cost of a single RDE. The number of patients who underwent undilated examination was estimated by random medical record review. The additional cost of a single RDE was determined from estimated examination times and payroll costs. Among patients without risk factors, 38 were identified for whom undilated examination rather than RDE had been performed. If prevention had been 10% effective, the substitution of 50,000 RDEs for undilated examinations costing the provider $433,000 would have been required per prevented case. These results suggest that most peripheral retinal diseases cannot be prevented by RDE. Routine dilated examination is an expensive test per prevented case. Published clinical guidelines lack evidence to recommend its use.