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Cost effectiveness of the type II Boston keratoprosthesis.

ABSTRACT: PURPOSE: Despite demonstrated cost effectiveness, not all corneal disorders are amenable to type I Boston keratoprosthesis (KPro) implantation. This includes patients with autoimmune diseases, such as Stevens-Johnson syndrome/toxic epidermal necrolysis. Type II KPro is implanted through the eyelids in severe dry eye and cicatricial diseases, and its cost effectiveness was sought. PATIENTS AND METHODS: In a retrospective chart review, 29 patients who underwent type II KPro surgery at the Massachusetts Eye and Ear Infirmary between the years 2000 and 2009 were identified. A total of 11 patients had 5-year follow-up data. Average cost effectiveness was determined by cost-utility analysis, comparing type II KPro surgery with no further intervention. RESULTS: Using the current parameters, the cost utility of KPro from third-party insurer (Medicare) perspective was 63,196 $/quality-adjusted life year . CONCLUSION: Efforts to refer those less likely to benefit from traditional corneal transplantation or type I KPro, for type II KPro surgery, may decrease both patient and societal costs.

SUBMITTER: Ament JD

PROVIDER: S-EPMC3178310 | biostudies-other | 2011 Mar

REPOSITORIES: biostudies-other

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Project description:STUDY DESIGN:Cost-effectiveness analysis. OBJECTIVE:To examine the cost-effectiveness of operative versus non-operative treatment of type-II odontoid fractures in patients older than 64 years old. SUMMARY OF BACKGROUND DATA:Significant controversy exists regarding the optimum treatment of geriatric patients with type-II odontoid fractures. Operative treatment leads to lower rates of non-union but carries surgical risks. Non-operative treatment does not carry surgical risks but has higher non-union rates. METHODS:A decision-analytic model was created to compare operative and non-operative treatment of type-II odontoid fractures among three age cohorts (65-74, 75-84, >84) based on expected costs, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs; cost per QALY gained). Age-specific mortality rates for both treatments, costs for treatment, and complication rates were taken from the literature, and data from 2010 US life tables were used for age-specific life expectancy. Costs of complications were estimated using data obtained at a level-I trauma center using micro-costing. Sensitivity analyses of all model parameters were conducted. RESULTS:Among the 65- to 74-year-old cohort, operative treatment was more costly ($53,407 vs. $30,553) and more effective (12.00 vs. 10.11 QALY), with an ICER of $12,078/QALY. Among the 75- to 84-year-old cohort, operative treatment was more costly ($51,308 vs. $29,789) and more effective (6.85 vs. 6.31 QALY), with an ICER of $40,467/QALY. Among the over-84 cohort, operative treatment was dominated by non-operative treatment as it was both more costly ($45,978 vs. $28,872) and less effective (2.48 vs. 3.73 QALY). The model was robust to sensitivity analysis across reasonable ranges for utility of union, disutility of complications and delayed surgery, and probabilities of non-union and complications. CONCLUSION:Operative treatment is cost-effective in patients age 65 to 84 when using $100,000/QALY as a benchmark but less effective and more costly than non-operative treatment in patients older than 84 years. LEVEL OF EVIDENCE:2.

Project description:Background and objectiveNearly all patients with multiple myeloma undergo multiple rounds of therapy. The phase III BOSTON trial of once-weekly selinexor and once-weekly bortezomib with dexamethasone (XVd) vs twice-weekly bortezomib and dexamethasone (Vd) is the basis for this cost-effectiveness analysis in previously treated multiple myeloma from a US commercial payer perspective over a lifetime horizon.MethodsA partitioned survival model enabled use of direct overall survival and progression-free survival curves from BOSTON to generate four health states for XVd and Vd: progression-free survival on treatment, progression-free survival off treatment, post-progression, and mortality. Using a 1-week cycle length, benefits and costs were discounted at 3.0% annually. Additional comparators were included in an exploratory analysis that compared XVd against seven additional regimens (six triplets, one doublet).ResultsAfter considering costs, utility, progression, and survival, the base-case incremental cost-effectiveness ratio of XVd vs Vd was $475,430/quality-adjusted life-year (QALY). The 50% cost-effectiveness probability midpoint was near $470,000/QALY, based on a probabilistic sensitivity analysis. The robustness of the analysis was supported by additional scenario assessment and deterministic and probabilistic sensitivity analyses, which generally demonstrated little variance, with greatest sensitivity to variations in discount rates and utility values. In an exploratory analysis against external comparators, XVd showed a higher QALY gain with a lower cost (i.e., dominance) compared with lenalidomide/dexamethasone (Rd), pomalidomide/bortezomib/dexamethasone (PVd), and carfilzomib/pomalidomide/dexamethasone (KPd).ConclusionsAddition of XVd to the previously treated multiple myeloma treatment landscape provides a novel oral treatment option, which, when compared to Vd in the base-case analysis resulted in an incremental cost-effectiveness ratio of $475,430/QALY. Exploratory analyses comparing against external comparators suggest that XVd was dominant vs Rd, PVd, and KPd.

Project description:OBJECTIVE: To evaluate the cost-effectiveness of enzyme replacement therapy (ERT) compared to standard medical care without ERT in the Dutch cohort of patients with type 1 Gaucher disease (GD I). DESIGN: Cost-effectiveness analysis was performed using a life-time state-transition model of the disease's natural course. Transition probabilities, effectiveness data and costs were derived from retrospective data and prospective follow-up of the Dutch study cohort. SETTING: The tertiary referral center for Gaucher disease in the Netherlands. PARTICIPANTS: The Dutch cohort of patients with GD I. INTERVENTION: ERT versus standard medical care without ERT in symptomatic patients. MAIN OUTCOME MEASURES: Years free of end organ damage (YFEOD) (splenectomy, bone complication, malignancy, multiple complications), quality adjusted life years (QALY), and costs. RESULTS: Over an 85 year lifetime, an untreated GD I patient will generate 48.9 YFEOD and 55.86 QALYs. Starting ERT in a symptomatic patient increases the YFEOD by 12.8 years, while the number of QALYs gained increases by 6.27. The average yearly ERT medication costs range between € 124,000 and € 258,000 per patient. The lifetime costs of ERT starting in the symptomatic stage are € 5,716,473 against € 171,780 without ERT, a difference of € 5,544,693. Consequently, the extra costs per additional YFEOD or per additional QALY are € 434,416 and € 884,994 respectively. After discounting effects by 1.5% and costs by 4% and under a reasonable scenario of ERT unit cost reduction by 25%, these incremental cost-effectiveness ratios could decrease to € 149,857 and € 324,812 respectively. DISCUSSION: ERT is a highly potential drug for GD I with substantial health gains. The conservatively estimated incremental cost-effectiveness ratios are substantially lower than for Pompe and Fabry disease. We suggest that the high effectiveness has contributed importantly to acceptance of reimbursement of ERT for GD I. The present study may further support discussions on acceptable price limits for ultra-orphan products.

Project description:Purpose Patients who are diagnosed with stage IV metastatic melanoma have an estimated 5-year relative survival rate of only 17%. Randomized controlled trials of recent US Food and Drug Administration-approved immune checkpoint inhibitors-pembrolizumab (PEM), nivolumab (NIVO), and ipilumumab (IPI)-demonstrate improved patient outcomes, but the optimal treatment sequence in patients with BRAF wild-type metastatic melanoma remains unclear. To inform policy makers about the value of these treatments, we developed a Markov model to compare the cost-effectiveness of different strategies for sequencing novel agents for the treatment of advanced melanoma. Materials and Methods We developed Markov models by using a US-payer perspective and lifetime horizon to estimate costs (2016 US$) and quality-adjusted life years (QALYs) for treatment sequences with first-line NIVO, IPI, NIVO + IPI, PEM every 2 weeks, and PEM every 3 weeks. Health states were defined for initial treatment, first and second progression, and death. Rates for drug discontinuation, frequency of adverse events, disease progression, and death obtained from randomized phase III trials were used to determine the likelihood of transition between states. Deterministic and probabilistic sensitivity analyses were conducted to evaluate model uncertainty. Results PEM every 3 weeks followed by second-line IPI was both more effective and less costly than dacarbazine followed by IPI then NIVO, or IPI followed by NIVO. Compared with the first-line dacarbazine treatment strategy, NIVO followed by IPI produced an incremental cost effectiveness ratio of $90,871/QALY, and first-line NIVO + IPI followed by carboplatin plus paclitaxel chemotherapy produced an incremental cost effectiveness ratio of $198,867/QALY. Conclusion For patients with treatment-naive BRAF wild-type advanced melanoma, first-line PEM every 3 weeks followed by second-line IPI or first-line NIVO followed by second-line IPI are the most cost-effective, immune-based treatment strategies for metastatic melanoma.

Project description:IntroductionBT-001 (AspyreRx™) prescription digital therapy, a form of personalized cognitive behavioral therapy, has demonstrated clinically meaningful and durable hemoglobin A1c reductions in patients with type 2 diabetes (T2D). The current study examined the cost-effectiveness of BT-001 plus standard of care (SoC) versus SoC alone in T2D over a lifetime horizon from a healthcare payer perspective.MethodsWe modeled the T2D pathway using an individual patient-level simulation; clinical data were sourced from the intention-to-treat subset of the BT-001 randomized clinical trial (RCT). SoC across both arms included the composition of oral and injectable treatments for T2D. Events were simulated using the United Kingdom Prospective Diabetes Study Outcomes Model 2 risk equation. A 3-month model cycle length was used in the first year, then annual model cycles were used in line with the original risk engine specifications. Patient characteristics informed event equations and Monte Carlo random sampling was used to assess the occurrence of events within each model cycle. Incidence of hypoglycemic events, drug discontinuation, costs, and health utilities and disutility values were sourced from the literature.ResultsFrom a payer perspective, BT-001 plus SoC versus SoC alone was dominant with a gain in quality-adjusted life years (QALYs) of 0.101 and cost savings of $7343 per patient over the lifetime horizon (i.e., more effective and less costly). BT-001 plus SoC was cost-effective at a willingness-to-pay of $100,000 per QALY (incremental net monetary benefit was $17,443). Savings with BT-001 were primarily driven by a reduction in drug acquisition costs. The reduction in hemoglobin A1c with BT-001 was associated with fewer T2D complications.ConclusionsBT-001 plus SoC was estimated to dominate SoC alone over the lifetime horizon from a payer perspective, suggesting that using BT-001 can empower patients to better manage their diabetes with the potential for lifelong advantages.

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Cost effectiveness of the type II Boston keratoprosthesis.

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