A cost-effectiveness analysis of iStent inject combined with phacoemulsification cataract surgery in patients with mild-to-moderate open-angle glaucoma in France.

OBJECTIVE: To investigate the cost-effectiveness of implementing iStent inject trabecular bypass stent (TBS) in conjunction with cataract surgery (Cat Sx) in patients with mild-to-moderate glaucoma from a societal perspective in France. The secondary objective was to explore the economic impact of iStent inject TBS in patients who comply to different degrees with their anti-glaucoma medications. METHODS: A previously published Markov model was adapted to estimate the cost-effectiveness of treatment with iStent inject TBS + Cat Sx versus Cat Sx alone over a lifetime time horizon in patients with mild-to-moderate open-angle glaucoma in France. Progression was modeled by health states reflecting increasing stages of vision loss. Disease progression was obtained from the two-year randomized clinical trial assessing safety and effectiveness of both interventions. French specific health-state utilities and costs were obtained through a targeted literature review. Model structure and inputs were validated by French ophthalmologists. Outcomes were expressed as incremental cost per quality-adjusted life-year (QALY) gained. The robustness of results was tested through sensitivity analyses. RESULTS: iStent inject TBS + Cat Sx reduced the number of medications needed and risk of blindness. Incremental cost and QALYs were €75 and 0.065 leading to an incremental cost-effectiveness ratio (ICER) of €1,154/QALY gained. ICER ranged from dominating for non-persistent patients to €31,127 patients fully persistent with their medication regime. Results from one-way sensitivity analysis had a maximum ICER of €29,000 when varying input parameters. iStent inject TBS + Cat Sx had an 86% chance of being cost-effective at a willingness-to-pay threshold of €30,000 per QALY gained. CONCLUSION: Results demonstrate that iStent inject TBS + Cat Sx is a cost-effective intervention for intraocular pressure reduction when compared to Cat Sx alone in France.

Determinant Factors of the Direct Medical Costs Associated with Genotype 1 Hepatitis C Infection in Treatment-Experienced Patients.

OBJECTIVE: Limited evidence is available on predictors of medical resource utilization (MRU) and related direct costs, especially in treatment-experienced patients infected with genotype 1 hepatitis C virus (HCV). This study aimed at investigating patient and treatment characteristics that predict MRU and related non-drug costs in treatment-experienced patients with chronic hepatitis C (CHC) treated with simeprevir (SMV) or telapravir (TVR) in combination with pegylated interferon and ribavirin (PegIFN/R). PATIENTS AND METHODS: A total of 709 patients who completed the 72-week ATTAIN trial were included in the study. Cost data were analysed from the UK NHS perspective. Descriptive statistics and regression analyses were used to determine patterns and predictors of total MRU-related costs associated with SMV/PegIFN/R and TVR/PegIFN/R. RESULTS: Independent predictors for total MRU-related costs were age, region and the following interaction terms: (1) gender × F3-F4 METAVIR score × baseline viral load (BLVL), (2) body mass index (BMI) × F3-F4 METAVIR score × prior response to PegIFN/R and (3) gender × achievement of SVR at 12 weeks (SVR12) × BLVL. A F3-F4 METAVIR score was a stronger predictor of total MRU-related costs than SVR12. Predictors of adverse events included older age, female gender, low BMI, TVR/PegIFN/R and SVR12. Wilcoxon rank sum test revealed comparable total MRU-related costs between SMV/PegIFN/R and TVR/PegIFN/R. CONCLUSION: To the best of our knowledge, this study is the first to describe the relationship between commonly admitted predictors of MRU-related costs and their joint effect on total MRU-related costs in treatment-experienced patients with CHC. The identified predictors of MRU-related costs suggest that significant treatment costs can be avoided by starting treatment early before the disease progresses. Furthermore, adverse events seem to be the most important factor to take into consideration for the choice of treatment, especially when therapeutic options are associated with similar levels of medical resource utilization and associated costs.

Predicting the impact of adverse events and treatment duration on medical resource utilization-related costs in hepatitis C genotype 1 treatment-naïve patients receiving antiviral therapy.

OBJECTIVES: Studies on medical resource utilization (MRU) and related costs are important for evaluating the potential patient management and cost-effectiveness implications of antiviral treatments for hepatitis C virus (HCV) infection. The objectives of this study were (i) to compare the MRU and related costs for two treatment approaches; (ii) to identify the main drivers of resource use and costs; and (iii) to assess the effects of various treatment regimen attributes on MRU-related costs in a UK clinical setting. METHODS: The analysis used data collected alongside the simeprevir (SMV) phase III trials for treatment-naïve genotype 1 HCV-infected patients; these data covered outpatient consultations with specialists, emergency room visits and hospital admissions. Logistic regressions were constructed to estimate the predictors of resource utilization, and a two-part multivariable analysis model was used to determine the total costs of treatment in the UK. RESULTS: Data on 731 patients receiving SMV plus pegylated interferon and ribavirin (SMV/PegIFN/R) or PegIFN/R were included in the analysis. While MRU was similar between the SMV and PegIFN/R groups, MRU-related costs were significantly lower in the SMV group than in the PegIFN/R group (P < 0.05). High body mass index (P < 0.05), severe fibrosis (P < 0.05), shortened treatment duration to 24 weeks (P < 0.05), and anaemia and rash during treatment (P < 0.001) were identified as predictors of hospitalization and outpatient visits and as drivers of total costs. Univariate sensitivity analyses suggested that shortened treatment duration and lower occurrence of rash lead to large cost savings. CONCLUSION: This study identified both baseline and on-treatment antiviral therapy characteristics as drivers of MRU-related costs for HCV patients following antiviral therapy. The shortened treatment duration and reduction in rash due to treatment with SMV triple therapy lead to substantial non-drug cost savings, compared with PegIFN/R treatment. This suggests that there are potential patient management and cost-effectiveness implications associated with the choice of specific antiviral treatments.

Cost-effectiveness of rivaroxaban versus warfarin for stroke prevention in atrial fibrillation in the Belgian healthcare setting.

BACKGROUND: Warfarin, an inexpensive drug that has been available for over half a century, has been the mainstay of anticoagulant therapy for stroke prevention in patients with atrial fibrillation (AF). Recently, rivaroxaban, a novel oral anticoagulant (NOAC) which offers some distinct advantages over warfarin, the standard of care in a world without NOACs, has been introduced and is now recommended by international guidelines. OBJECTIVE: The aim of this study was to evaluate, from a Belgian healthcare payer perspective, the cost-effectiveness of rivaroxaban versus use of warfarin for the treatment of patients with non-valvular AF at moderate to high risk. METHODS: A Markov model was designed and populated with local cost estimates, safety-on-treatment clinical results from the pivotal phase III ROCKET AF trial and utility values obtained from the literature. RESULTS: Rivaroxaban treatment was associated with fewer ischemic strokes and systemic embolisms (0.308 vs. 0.321 events), intracranial bleeds (0.048 vs. 0.063), and myocardial infarctions (0.082 vs. 0.095) per patient compared with warfarin. Over a lifetime time horizon, rivaroxaban led to a reduction of 0.042 life-threatening events per patient, and increases of 0.111 life-years and 0.094 quality-adjusted life-years (QALYs) versus warfarin treatment. This resulted in an incremental cost-effectiveness ratio of €8,809 per QALY or €7,493 per life-year gained. These results are based on valuated data from 2010. Sensitivity analysis indicated that these results were robust and that rivaroxaban is cost-effective compared with warfarin in 87 % of cases should a willingness-to-pay threshold of €35,000/QALY gained be considered. CONCLUSIONS: The present analysis suggests that rivaroxaban is a cost-effective alternative to warfarin therapy for the prevention of stroke in patients with AF in the Belgian healthcare setting.

Implications of a data-driven approach to treatment with growth hormone in children with growth hormone deficiency and Turner syndrome.

BACKGROUND: Response to growth hormone (GH) therapy may vary between individual patients. Therefore the use of GH in children should be closely monitored to avoid over, under, or ineffective treatment regimens. The treatment response can be evaluated using growth prediction models. In an effort to improve the accuracy of these prediction models, Ranke et al. (J Clin Endocrinol Metab 95(3):1229-37) proposed a novel 'data-driven' approach based on a quantitative analysis of a large cohort of patients from the Pfizer International Growth Database (KIGS) treated with Genotropin (human growth hormone). This model allows physicians to predict and evaluate the level of growth response and responsiveness for their patients so they can adapt treatment accordingly. By comparing the actually observed and the predicted growth response the ability of an individual to respond to GH (responsiveness) can be estimated and further treatment can be adapted accordingly OBJECTIVE: To determine the potential population level reduction in the amount of GH used and impact on height outcome of using this data-driven approach to guide treatment decisions, compared to conventional, 'experience-based' GH treatment in prepubertal patients with growth hormone deficiency (GHD) or Turner syndrome (TS). METHODS: A model was developed to study the height outcome and the total amount of GH used in the presence or absence of data-driven treatment decisions. The proportion of patients for whom height outcome could be improved or GH use could be reduced (i.e. for low compliance, high or low responder) was estimated using the KIGS cohort. The analysis assumed that this segmentation allows physicians to tailor dosage to the individual patient's needs or even to discontinue therapy when it is not effective. The analysis used a 4-year time horizon, with Germany as an example country, but results are extendable to other countries. Only the total amount of GH used was included, and effects were defined as the height outcome after 4 years. RESULTS: The analysis estimated that an evidence-driven approach may reduce the total amount of GH utilized by 7.0 % over 4 years for the treatment of short stature in prepubertal patients with GHD and TS in Germany. Despite the reduction in drug use the average growth outcomes remained unaffected with the new treatment approach. Univariate and probabilistic sensitivity analyses showed that the results are robust. CONCLUSIONS: Our analysis showed that using a data-driven approach to guide treatment decisions for children with GHD or TS is estimated to result in efficiencies in the amount of GH used, without reducing the average growth in the population.

The economic impact and cost-effectiveness of urinary neutrophil gelatinase-associated lipocalin after cardiac surgery.

BACKGROUND: Acute kidney injury (AKI) is common after cardiac surgery, and expeditious recognition with specific biomarkers may help improve outcome. OBJECTIVE: Because the economic impact of a biomarker-based diagnostic strategy is unknown, we assessed the cost-effectiveness of using urinary neutrophil gelatinase-associated lipocalin (NGAL) for the diagnosis of AKI after cardiac surgery compared with current diagnostic methods. METHODS: A decision analysis model was developed using the societal perspective to evaluate the cost-effectiveness of NGAL. Cost per quality-adjusted life-year (QALY) was determined for NGAL and standard strategies. The base case was a 67-year-old male patient undergoing coronary artery bypass graft surgery in the United Kingdom. Multiple sensitivity analyses were performed to determine how cost-effectiveness would vary with changes in the underlying clinical and economic variables. RESULTS: The base case yielded expected costs of £4244 and 11.86 QALYs for the NGAL strategy compared with £4672 and 11.79 QALYs for the standard therapy. The cost-effectiveness ratio for the NGAL strategy was £358/QALY compared with £396/QALY for the standard regimen. Cost-effectiveness increased as the treatment effect-defined as the ability to prevent progression of established AKI (kidney injury or failure)-for the therapy triggered by an elevated NGAL level rose. Sensitivity analysis demonstrated that the model was most responsive to the probability of developing AKI and least sensitive to the test cost for NGAL. Probabilistic sensitivity analysis supported the NGAL strategy as the most cost-effective option. Because this study was a decision analysis model incorporating a nonspecific treatment for AKI (as opposed to an observational study or controlled trial), model structural assumptions may therefore have underestimated mortality and the likelihood of developing AKI, although these were tested in multiple sensitivity analyses. Indirect costs were also not explicitly factored. CONCLUSION: The use of urinary NGAL after cardiac surgery appears to be cost-effective in the early diagnosis of AKI.