Cost Effectiveness of Fremanezumab in Episodic and Chronic Migraine Patients from a Japanese Healthcare Perspective.

BACKGROUND AND OBJECTIVES: Fremanezumab is an effective treatment for episodic (EM) and chronic migraine (CM) patients in Japan, but its cost effectiveness remains unknown. The objective of this study was to determine the cost effectiveness of fremanezumab compared with standard of care (SOC) in previously treated EM and CM patients from a Japanese healthcare perspective. METHODS: Estimated regression models were implemented in a probabilistic Markov model to inform effectiveness and health-related quality-of-life data for fremanezumab and SOC. The model was further populated with data from the literature. The adjusted Japanese healthcare perspective included productivity losses. The main model outcomes were quality-adjusted life-years (QALYs), costs (2022 Japanese Yen [¥]), and incremental outcomes including the incremental cost-effectiveness ratio (ICER). Analyses were performed separately for the EM and CM patients and combined. Costs and effects were discounted at an annual rate of 2.0%. RESULTS: The mean QALYs over a 25-year time horizon for the EM and CM populations combined were 13.03 for SOC and 13.15 for fremanezumab. The associated costs were ¥27,550,292 for SOC and ¥28,371,048 for fremanezumab. QALYs were higher and costs lower for EM patients compared with CM patients for both fremanezumab and SOC. The deterministic ICERs of fremanezumab versus SOC were ¥6,334,861 for EM, ¥7,393,824 for CM, and ¥6,530,398 for EM and CM combined. Indirect costs and choice of mean migraine days model distribution had a substantial impact on the ICER. CONCLUSION: Using fremanezumab in a heterogeneous mixture of Japanese EM and CM patients resulted in a reduction of monthly migraine days and thus more QALYs compared with SOC. The cost effectiveness of fremanezumab versus SOC in EM and CM patients resulted in an ICER of ¥6,530,398, from an adjusted Japanese public healthcare perspective.

Fremanezumab is an effective treatment for episodic and chronic migraine patients in Japan, but it is unknown how the costs relate to the health benefits. The current research determined the relation between costs and effects of fremanezumab compared with the current standard of care in Japanese clinical practice, to see if the costs are justified by the health benefits. A model was used to inform the treatment effect of fremanezumab and standard of care. Data on costs, the frequency in which health care was used, and impairment of work due to migraine were also included in the model and obtained from the literature. The main outcomes were the number of years that patients were alive while taking their quality of life into account, costs, and the difference in these outcomes between patients who were treated with fremanezumab and those receiving standard of care. Subsequently, it was estimated how costs and effects related to one another and whether the costs were justified by the health benefits. The outcomes showed that patients treated with fremanezumab had a better quality of life compared with those receiving standard of care, while the costs associated with fremanezumab were higher. Compared with standard of care, the health benefits of treating patients with fremanezumab were justified by the costs within an acceptable range. Taking the absence from work due to illness into account had a substantial impact on the model outcomes.

Long-Term Outcomes of Eladocagene Exuparvovec Compared with Standard of Care in Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency: A Modelling Study.

INTRODUCTION: Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare disease with symptoms including movement disorders, developmental delays, and autonomic symptoms starting from birth; further, patients with AADC deficiency are at a high risk of death in the first decade of life. Limited information on the impact of treatment with gene therapy on patients' disease trajectories and survival, quality-of-life, and resource usage benefits are available. METHOD: A cohort-based model with a lifetime horizon has been developed, based on motor milestones, to estimate the long-term benefits for patients after treatment with eladocagene exuparvovec compared to best supportive care (BSC). The model takes a National Health Service (NHS) perspective using a UK setting. The model comprises two parts: the developmental phase, in which patients with initially no motor function can progress to other motor milestone states, and a long-term projection phase. Efficacy for eladocagene exuparvovec is derived from clinical trial data with a duration up to 120 months. As the incidence of AADC deficiency is low, data for key model inputs is lacking; therefore estimates of survival by motor milestone were based on proxy diseases. A disease-specific utility study provided quality of life inputs and a burden of illness study informed inputs for disease management. RESULTS: The model indicates survival (25.25 undiscounted life years gained) and quality-of-life benefits (20.21 undiscounted quality-adjusted life years [QALYs] gained) for patients treated with eladocagene exuparvovec compared to BSC. Resource usage costs are greater for patients treated with eladocagene exuparvovec, mainly due to the increased life expectancy during which patients accrue additional healthcare resource usage. Scenario analyses indicate robust results. CONCLUSION: This study assessed long-term outcomes for patients with AADC deficiency. Patients treated with eladocagene exuparvovec were found to have improved survival and quality of life benefits compared to patients treated with BSC.

Modeling Monthly Migraine-Day Distributions Using Longitudinal Regression Models and Linking Quality of Life to Inform Cost-Effectiveness Analysis: A Case Study of Fremanezumab in Japanese-Korean Migraine Patients.

BACKGROUND AND OBJECTIVES: As regression approaches have been used more recently to model the effectiveness and health-related quality of life (HRQOL) of novel migraine treatments, an example is provided for fremanezumab. The objective is to estimate the distribution of mean monthly migraine days (MMD) as a continuous variable and corresponding migraine-specific utility values as a function of the MMD, to inform health states in a cost-effectiveness model (CEM). METHODS: Three longitudinal regression models (zero-adjusted gamma [ZAGA], zero-inflated beta-binomial [ZIBB], and zero-inflated negative binomial [ZINBI]) were fitted to Japanese-Korean clinical trial data of episodic (EM) and chronic migraine (CM) patients treated with fremanezumab or placebo, to estimate MMD over a period of 12 months. The EQ-5D-5L and the migraine-specific quality-of-life (MSQ), mapped to the EQ-5D-3L, questionnaires were used to measure HRQOL. Migraine-specific utility values were estimated as a function of MMD using a linear mixed effects model. RESULTS: The ZIBB models fitted the data best in estimating the distribution of mean MMD over time. MSQ-derived values were more sensitive than the EQ-5D-5L values for the effect of the number of MMD on HRQOL, with higher values for less MMD and more time on treatment. CONCLUSIONS: Using longitudinal regression models to estimate MMD distributions and linking utility values as a function is an appropriate method to inform CEMs and capture inter-patient heterogeneity. The observed distribution shifts demonstrated fremanezumab's effect at reducing MMD for both EM and CM patients, while treatment effect on HRQOL was captured by MMD and time on treatment.

The current study provides an example of an approach that can be used to estimate the number of migraine days per month and the quality of life of migraine patients. The outcomes of this approach can give an impression of how well a patient reacts to a new migraine treatment called fremanezumab. In this study, different mathematical equations were used to measure the migraine days per month and quality of life over a period of 1 year. The data came from Japanese-Korean patients that participated in clinical trials. The patients reported the number of days that they had migraine and their quality of life was measured with two validated questionnaires. With the gathered data, the quality of life was calculated for the number of migraine days that a patient could have per month. Patients who had the fewest migraine days and were treated the longest with the new treatment reported the best quality of life. The investigated approach is an appropriate method to measure the impact of fremanezumab on the number of monthly migraine days and a patient's quality of life. The measurements of this approach can be linked to other parameters in an economic model to estimate the costs required to reach a certain level of treatment effect with this new migraine treatment.

Cost-effectiveness analysis of KTE-X19 CAR T therapy versus real-world standard of care in patients with relapsed/refractory mantle cell lymphoma post BTKi in England.

AIMS: The objective of this study is to estimate the cost-effectiveness of KTE-X19 versus standard of care (SoC) in the treatment of patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL) post-Bruton tyrosine kinase inhibitor (BTKi) treatment from a UK healthcare perspective. MATERIALS AND METHODS: A three-state partitioned survival model (pre-progression, post-progression and death) with a cycle length of one month was used to extrapolate progression-free and overall survival over a lifetime horizon. Population inputs along with KTE-X19 (brexucabtagene autoleucel) efficacy and safety data were derived from the single-arm trial ZUMA-2 (NCT02601313). The composition of SoC was informed by a literature-based meta-analysis, SoC efficacy data were obtained from the SCHOLAR-2 real-world study. Survival was modelled using standard parametric curves for SoC and a mixture-cure methodology for KTE-X19. It was assumed that patients whose disease had not progressed after five years experienced long-term remission. Costs, resource use and utility, and adverse event disutility inputs were obtained from published literature and publicly available data sources. An annual discount rate of 3.5% was applied to costs and health outcomes. Modelled outcomes for KTE-X19 and SoC included expected life years (LY), quality-adjusted life years (QALY) and total costs. Deterministic and probabilistic sensitivity analyses and scenario analyses were performed. RESULTS: Estimated median survival was 5.96 years for KTE-X19 and 1.38 for SoC. Discounted LYs, QALYs and lifetime costs were 8.27, 5.99 and £385,765 for KTE-X19 versus 1.98, 1.48 and £79,742 for SoC, respectively. The KTE-X19 versus SoC cost per QALY was £67,713 and the cost per LY was £48,645. Influential scenario analyses use alternative KTE-X19 survival curves and discount rates, and shorter time horizons. CONCLUSION: Considering the survival and quality of life benefits compared to SoC, KTE-X19 for R/R MCL appears as a cost-effective treatment in the real-world UK setting.

An Integrated Pharmacokinetic-Pharmacodynamic-Pharmacoeconomic Modeling Method to Evaluate Treatments for Adults with Schizophrenia.

INTRODUCTION: Schizophrenia is a chronic mental disorder that worsens with each relapse. Long-acting injectable (LAI) antipsychotics may prevent the exacerbation of symptoms and occurrence of relapses through improved continuity of care. Different dose regimens are available for the LAIs aripiprazole monohydrate (AM) and aripiprazole lauroxil (AL), but their cost effectiveness is unclear. OBJECTIVES: The study aim was to compare costs and effects (relapses) of the different aripiprazole LAI dose regimens to inform clinical and US payer decisions. METHODS: A state-transition model calculated the outcomes of eight LAI dose regimens based on their relapse rates. As effectiveness data from randomized controlled trials were unavailable, relapse rates were modeled using pharmacokinetic and pharmacodynamic evidence. These described blood plasma levels of aripiprazole as a function of AM and AL dose regimens and described the probability of relapse as a function of aripiprazole blood plasma levels. The analysis had a time horizon of 1 year and took the US healthcare payer perspective. The incremental cost per relapse avoided and the probability of cost effectiveness were calculated in deterministic and probabilistic analyses. Scenario analyses explored the model's main assumptions, and results were validated against external data and other cost-effectiveness analyses. RESULTS: Monthly administration of AM 400 mg consistently yielded the lowest predicted number of relapses across deterministic, probabilistic, and scenario analyses. The costs of treatment and relapses were projected to be the lowest with a monthly administration of AL 441 mg. The incremental cost per relapse avoided with AM 400 mg ranged from AM 400 mg being dominant to $US83,300. From willingness-to-pay thresholds of $US30,000 per relapse avoided, the probability of cost effectiveness was highest for AM 400 mg. The validation showed alignment with external data. CONCLUSION: The analysis highlighted the robustness of the novel framework based on pharmacokinetic and pharmacodynamic evidence and demonstrated an application in a postmarketing setting.

Cost-effectiveness and budget impact of venetoclax in combination with rituximab in relapsed/refractory chronic lymphocytic leukemia in Switzerland.

INTRODUCTION: Venetoclax in combination with rituximab (VEN + R) demonstrated prolonged overall survival (OS) and progression-free survival (PFS) for patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) in comparison to standard chemoimmunotherapy [bendamustine + rituximab (BR)]. We conducted a cost-effectiveness and budget impact analysis comparing VEN + R versus six comparators from the Swiss healthcare payer perspective. METHODS: A three-state partitioned survival model, developed in accordance with NICE and ISPOR decision modelling guidelines, was adapted to Switzerland. Model inputs were informed by the MURANO trial (survival data, patient characteristics), publicly available Swiss sources (drug prices, inpatient and outpatient costs), Swiss National Institute of Cancer Epidemiology and Registration data (incidence and prevalence values), and Swiss medical expert feedback. We used published (dis-)utility values and adverse event probabilities. RESULTS: Over a lifetime, VEN + R resulted in an expected gain of 2.60 quality-adjusted life years (QALYs) per patient and incremental costs of Swiss Francs (CHF) 147,851 compared to BR, leading to an incremental cost-effectiveness ratio of CHF 56,881/QALY gained. Other treatment strategies (for example ibrutinib versus VEN + R) resulted in higher costs and lower QALYs. Results were not different for subgroups of patients with/without deletion of chromosome 17p/tumour protein 53 mutation. In scenario analysis, changes in post-progression treatment costs demonstrated a high impact on results. We estimated an expected value of perfect information of CHF 3,318/patient. A moderate VEN + R uptake was estimated to save CHF 12.3 million during 5 years. CONCLUSIONS: Using a threshold of CHF 100,000 per QALY, VEN + R was projected to be cost-effective vs BR.

Cost-effectiveness for KTE-X19 CAR T therapy for adult patients with relapsed/refractory mantle cell lymphoma in the United States.

AIMS: The objective of this study is to estimate the cost-effectiveness of KTE-X19 versus standard of care (SoC) in the treatment of relapsed/refractory (R/R) mantle cell lymphoma (MCL) patients from a US healthcare perspective. MATERIALS AND METHODS: A three-state partitioned-survival model (pre-progression, post-progression, and death) with a cycle length of 1 month was used to extrapolate progression-free and overall survival (OS) over a lifetime horizon. Due to the long tail of the OS curve, OS was modeled applying a mixture-cure methodology, using the assumption that patients whose disease had not progressed after 5 years experienced long-term remission. Population inputs were derived from the ZUMA-2 trial. This was also the source of KTE-X19 efficacy and safety data, while this data was obtained from the literature for SoC. Costs and resource use inputs were derived from the published literature and publicly available data sources. Health state utilities were derived from the ZUMA-2 trial (NCT02601313), applying the US tariff. Adverse event disutilities were derived from the published literature. Costs and health outcomes were discounted at 3% per year. The model estimated expected life years (LY), quality-adjusted life years (QALY), and total costs for KTE-X19 vs SoC. Deterministic and probabilistic sensitivity analyses were performed. RESULTS: Median survival was 9.71 years for KTE-X19 and 2.13 for SoC. Discounted LYs, QALYs, and lifetime costs were 8.99, 7.39, and $693,832 for KTE-X19 vs 4.47, 3.65, and $574,263 for SoC, respectively. The KTE-X19 vs SoC cost per QALY was $31,985. The most influential model parameter was the utility for patients with long-term remission. At a willingness-to-pay threshold of $150,000 per QALY, the probability that KTE-X19 was cost-effective was 99%. CONCLUSION: The treatment of R/R MCL with KTE-X19 presents a potentially cost-effective alternative to the current SoC, deriving its value from incremental survival and health-related quality-of-life benefits.

Health State Utilities of Patients with Heart Failure: A Systematic Literature Review.

BACKGROUND AND OBJECTIVES: New treatments and interventions are in development to address clinical needs in heart failure. To support decision making on reimbursement, cost-effectiveness analyses are frequently required. A systematic literature review was conducted to identify and summarize heart failure utility values for use in economic evaluations. METHODS: Databases were searched for articles published until June 2019 that reported health utility values for patients with heart failure. Publications were reviewed with specific attention to study design; reported values were categorized according to the health states, 'chronic heart failure', 'hospitalized', and 'other acute heart failure'. Interquartile limits (25th percentile 'Q1', 75th percentile 'Q3') were calculated for health states and heart failure subgroups where there were sufficient data. RESULTS: The systematic literature review identified 161 publications based on data from 142 studies. Utility values for chronic heart failure were reported by 128 publications; 39 publications published values for hospitalized and three for other acute heart failure. There was substantial heterogeneity in the specifics of the study populations, methods of elicitation, and summary statistics, which is reflected in the wide range of utility values reported. EQ-5D was the most used instrument; the interquartile limit for mean EQ-5D values for chronic heart failure was 0.64-0.72. CONCLUSIONS: There is a wealth of published utility values for heart failure to support economic evaluations. Data are heterogenous owing to specificities of the study population and methodology of utility value elicitation and analysis. Choice of value(s) to support economic models must be carefully justified to ensure a robust economic analysis.

A Systematic Review of Medical Costs Associated with Heart Failure in the USA (2014-2020).

BACKGROUND: Heart failure presents a growing clinical and economic burden in the USA. Robust cost data on the burden of illness are critical to inform economic evaluations of new therapeutic interventions. OBJECTIVES: This systematic literature review of heart failure-related costs in the USA aimed to assess the quality of the published evidence and provide a narrative synthesis of current data. METHODS: Four electronic databases (MEDLINE, EMBASE, EconLit, and the Centre for Reviews and Dissemination York Database, including the NHS Economic Evaluation Database and Health Technology Assessment Database) were searched for journal articles published between January 2014 and March 2020. The review, registered with PROSPERO (CRD42019134201), was restricted to cost-of-illness studies in adults with heart failure events in the USA. RESULTS: Eighty-seven studies were included, 41 of which allowed a comparison of cost estimates across studies. The annual median total medical costs for heart failure care were estimated at $24,383 per patient, with heart failure-specific hospitalizations driving costs (median $15,879 per patient). Analyses of subgroups revealed that heart failure-related costs are highly sensitive to individual patient characteristics (such as the presence of comorbidities and age) with large variations even within a subgroup. Additionally, differences in study design and a lack of standardized reporting limited the ability to compare cost estimates. The finding that costs are higher for patients with heart failure with reduced ejection fraction compared with patients with preserved ejection fraction highlights the need for differentiating among different heart failure types. CONCLUSIONS: The review underpins the conclusion drawn in earlier reviews, namely that hospitalization costs are the key driver of heart failure-related costs. Analyses of subgroups provide a clearer understanding of sources of heterogeneity in cost data. While current cost estimates provide useful indications of economic burden, understanding the nuances of the data is critical to support its application.

Cost-effectiveness analysis of ocriplasmin versus watchful waiting for treatment of symptomatic vitreomacular adhesion in the US.

Aim: Evaluate the cost-effectiveness of ocriplasmin in symptomatic vitreomacular adhesion (VMA) with or without full-thickness macular hole ≤400 µm versus standard of care. Methods: A state-transition model simulated a cohort through disease health states; assignment of utilities to health states reflected the distribution of visual acuity. Efficacy of ocriplasmin was derived from logistic regression models using Ocriplasmin for Treatment for Symptomatic Vitreomacular Adhesion Including Macular Hole trial data. Model inputs were extracted from Phase III trials and published literature. The analysis was conducted from a US Medicare perspective. Results: Lifetime incremental cost-effectiveness ratio was US$4887 per quality-adjusted life year gained in the total population, US$4255 and US$10,167 in VMA subgroups without and with full-thickness macular hole, respectively. Conclusion: Ocriplasmin was cost effective compared with standard of care in symptomatic VMA.

Real-World Effectiveness and Real-World Cost-Effectiveness of Intravitreal Aflibercept and Intravitreal Ranibizumab in Neovascular Age-Related Macular Degeneration: Systematic Review and Meta-Analysis of Real-World Studies.

INTRODUCTION: Treatment of neovascular age-related macular degeneration (nAMD) has evolved with the advent of anti-vascular endothelial growth factor agents such that intravitreally administered aflibercept and ranibizumab (RBZ) have become the standard of care. Randomized clinical trials (RCTs) have demonstrated the benefits of these agents in nAMD; however, results achieved under RCT protocols may not always be replicated in clinical practice. Assessing real-world outcomes is important to estimate the effectiveness and cost-effectiveness of these two agents. Our objective was to assess the real-world effectiveness of intravitreally administered aflibercept and RBZ in treatment-naive patients with nAMD and determine the cost-effectiveness of intravitreally administered aflibercept versus RBZ in a real-world setting. METHODS: A multistage approach was undertaken. A systematic literature review (SLR) was completed to identify studies describing real-world outcomes in patients with nAMD treated intravitreally with aflibercept or RBZ. A meta-analysis of data identified in the SLR generated a pooled estimate of the effectiveness of intravitreally administered aflibercept and RBZ at 52 weeks and an estimate of treatment burden (injection frequency and monitoring). The impact of treatment effect modifiers, such as baseline visual acuity (VA) and age, were corrected through a multivariable meta-regression. A Markov state transition model was developed to estimate the real-world cost-effectiveness of intravitreally administered aflibercept using results from the pooled estimates for effectiveness and treatment burden as primary inputs. The analysis considered the perspective of the French National Healthcare System. RESULTS: Patients treated intravitreally with aflibercept had a mean age of 79.52 years and mean baseline VA of 55.80 Early Treatment Diabetic Retinopathy Study (ETDRS) letters. At week 52, mean VA gain was 5.30 ETDRS letters in patients reporting an average of 7.10 intravitreal injections of aflibercept and 8.65 visits (injection and/or monitoring). RBZ-treated patients were younger (77.28 years), with a lower mean baseline VA (52.81 ETDRS letters). At week 52, mean VA gain from baseline was 4.24 ETDRS letters, with an average of 5.88 injections and 10.10 visits (injection and/or monitoring). After correcting for differences in age (77.28 years) and baseline VA (52.81 ETDRS letters) and considering the current clinical practice with aflibercept and RBZ, the mean VA gain was 6.57 ETDRS letters for patients treated intravitreally with aflibercept and 4.42 ETDRS for patients treated intravitreally with RBZ. The cost-effectiveness analysis showed that intravitreally administered aflibercept is a more effective treatment option with an incremental gain in quality-adjusted life years (QALYs) (4.918 versus 4.880) and an incremental cost-effectiveness ratio (ICER) of €27,087 per QALY. CONCLUSIONS: The analysis identified differences in the overall treatment approach and how ophthalmologists use intravitreally administered aflibercept and RBZ in clinical practice. These differences ultimately influence the mean real-world effectiveness of the two agents. Intravitreal treatment with aflibercept (injection frequency and patients follow-up) was consistent and in line with the European label recommendations. Patients treated intravitreally with aflibercept in clinical practice reported a mean gain in VA of similar magnitude to the mean VA gain reported in the pivotal RCT. Conversely, treatment with RBZ varied significantly across the different studies. On average, RBZ-treated patients reported a low injection frequency and a frequent follow-up, driven in part by the high number of patients treated with pro re nata (PRN) regimens. RBZ-treated patients reported gains in VA versus baseline; however, the magnitude of the gain in VA was not comparable to the VA gains reported in the RBZ pivotal RCT. Intravitreal treatment with aflibercept was associated with better mean VA outcomes and an incremental gain in QALYs compared with RBZ and can be considered cost-effective for the treatment of nAMD in patients in France despite a higher price for each individual intravitreal injection of aflibercept compared with RBZ. FUNDING: Bayer AG, Basel.

Development of an Initial Conceptual Model of Multiple Myeloma to Support Clinical and Health Economics Decision Making.

Background. We aimed to develop and validate a conceptual model of multiple myeloma (MM) that characterizes the attributes affecting disease progression and patient outcomes, and the relationships between them. Methods. Systematic and targeted literature reviews identified disease- and patient-specific attributes of MM that affect disease progression and outcomes. These attributes were validated by a Delphi panel of four international MM experts, and a physician-validated model was constructed. Real-world clinical data from the Czech Registry of Monoclonal Gammopathies (RMG) was used to confirm the relationships between attributes using pairwise correlations and multiple Cox regression analysis. Results. The Delphi panel reached consensus that most cytogenetic abnormalities influenced disease activity, which results in symptoms and complications and affects overall survival (OS). Comorbidities and complications also affect OS. The entire panel agreed that quality of life was influenced by comorbidities, age, complications, and symptoms. Consensus was not reached in some cases, in particular, the influence of del(17p) on complications. The relationships between attributes were confirmed using pairwise analysis of real-world data from the Czech RMG; most of the correlations identified were statistically significant and the strength of the correlations changed with successive relapses. Czech RMG data were also used to confirm significant predictors of OS included in the model, such as age, Eastern Cooperative Oncology Group performance status, and extramedullary disease. Conclusions. This validated conceptual model can be used for economic modeling and clinical decision making. It could also inform the development of disease-based models to explore the impact of disease progression and treatment on outcomes in patients with MM.

Cost-effectiveness of ocriplasmin for the treatment of vitreomacular traction and macular hole.

BACKGROUND: If left untreated, vitreomacular traction (VMT) will infrequently improve through spontaneous resolution of vitreomacular adhesion (VMA), and patients remain at risk of further deterioration in vision. The mainstay of treatment for VMT is vitrectomy, an invasive procedure that carries the risk of rare but serious complications and further vision loss. As such, a 'watch and wait' approach is often adopted before this surgical intervention is performed. Ocriplasmin (microplasmin) is a potential alternative treatment for patients with symptomatic VMT that may remove the requirement for vitrectomy. OBJECTIVE: The purpose of this study was to evaluate the cost-effectiveness of ocriplasmin for the treatment of VMT in comparison to standard of care. STUDY DESIGN: A cohort-based computer simulation model was developed, capturing three mutually exclusive subgroups: 1) VMT without epiretinal membrane (ERM) or full thickness macular hole (FTMH), 2) VMT with ERM but no FTMH, and 3) VMT with FTMH. Transition probabilities between health states, utilities, and resource utilisation were estimated based on clinical trial results, the literature, and expert opinion. The cost per quality-adjusted life year (QALY) gained was estimated over a lifetime, using UK unit costs and utilities associated with visual acuity, adverse events, metamorphopsia, and surgical interventions. SETTING: Analyses were conducted from a UK payer perspective. POPULATION: Transition probabilities for the model were primarily estimated from patient-level data from the combined Phase 3 MIVI-TRUST trials in patients with symptomatic VMA/VMT, including when associated with a FTMH ≤400 µm. INTERVENTION: Ocriplasmin (microplasmin) is a one-time intravitreal injection designed specifically to release the abnormal traction between the macula and the vitreous and thereby treat VMT, as well as macular hole with persistent vitreous attachment. MAIN OUTCOME MEASURE: The main outcome measure of the economic evaluation was cost per QALY. RESULTS: In all subgroups, ocriplasmin management generated more QALYs: 1) VMT without ERM or FTMH (0.105, (0.036, 0.191)); 2) VMT with ERM but no FTMH (0.041, (0.011, 0.131)); and 3) VMT with FTMH (0.053, (-0.002, 0.113)). The initial treatment costs were partially offset by later savings and net costs were estimated at £1,901 (£1,325, £2,474), £2,491 (£1,067, £2,511), and £1,912 (£1,233, £2,506), respectively. Costs per QALY were estimated at £18,056 (£8,241, £64,874), £61,059 (£8,269, £168,664), and £36,250 (-£144,788, £290,338), respectively. Short-term efficacy parameters were found to be key drivers of results. CONCLUSION: Ocriplasmin is most cost-effective in VMT patients without either ERM or FTMH.