Cost-utility of new film-coated tablet formulation of deferasirox vs deferoxamine among major beta-thalassemia patients in Iran
Medicine (Baltimore). 2020;99(28):e20949
OBJECTIVES Thalassemia is a hereditary disease, which caused economic burden in developing countries. This study evaluated the cost utility of new formulation of deferasirox (Jadenu) vs deferoxamine (Desferal) among B-Thalassemia-major patients from payer perspective in Iran. METHODS An economic-evaluation through Markov model was performed. A systematic review was conducted in order to evaluate the clinical effectiveness of comparators. Because of chelating therapy is weight-dependent, patients were assumed to be 2 years-old at initiation in first and 18 years-old in second scenario, and model was estimated lifetime costs and utilities. Costs were calculated to the Iran healthcare system through payer perspective and measured effectiveness using quality-adjusted life years (QALYs). One-way sensitivity analysis and budget impact analysis was also employed. RESULTS The 381 studies were retrieved from systematic searching through databases. After eliminating duplicate and irrelevant studies, 2 studies selected for evaluating the effectiveness. Jadenu was associated with an incremental cost-effectiveness ratio (ICER) of 1470.6 and 2544.7 US$ vs Desferal in first and second scenario respectively. The estimated ICER for Jadenu compared to generic deferoxamine was 2837.0 and 6924.1 US$ for first and second scenario respectively. For all scenarios Jadenu is presumed as cost-effective option based on calculated ICER which was lower than 1 gross domestic product per capita in Iran. Sensitivity analysis showed that different parameters except discount rate and indirect cost did not have impact on results. Based on budget impact analysis the estimated cost for patients using Desferal (based on the market share of brand) was 44,021,478 US$ in 3 years vs 42,452,606 US$ in replacing 33% of brand market share with Jadenu. This replacement corresponded to the cost saving of almost 1,568,872 US$ for the payers in 3 years. The calculated cost of using generic deferoxamine in all patients was 68,948,392 US$. The increase in the cost of using Jadenu for 10% of all patients in this scenario would be 934,427 US$ (1.36%) US$ at the first year. CONCLUSIONS Based on this analysis, film-coated deferasirox appeared to be cost-effective treatment in comparison with Desferal for managing child and adult chronic iron overload in B-thalassemia major patients of Iran.
Ferric carboxymaltose for patients with heart failure and iron deficiency in Italy: cost-effectiveness and budget impact
Journal of comparative effectiveness research. 2019
Aim: To evaluate the cost-effectiveness of intravenous ferric carboxymaltose (FCM) versus placebo for the management of iron deficiency in patients with chronic heart failure in the Italian healthcare system and to estimate its impact on the national healthcare budget. Materials & methods: A Markov model was developed to project costs and health outcomes over 1 year, based on data from literature. Healthcare resources consumption was derived from an e-survey administered to clinicians. Costs were obtained from official tariffs. Results: Treatment with FCM represents a dominant strategy compared with placebo, leading to national budget annual savings of 20-97 million Euros, according to different increasing utilization rates. Conclusion: FCM is a cost-saving option for the treatment of chronic heart failure patients with iron deficiency in Italy.
Chronic heart failure (CHF) patients in the Italian healthcare system.
Intravenous ferric carboxymaltose (FCM) for the management of iron deficiency. A Markov model was developed to project costs and health outcomes over 1 year.
Treatment with FCM represents a dominant strategy compared with placebo, leading to national budget annual savings of 20-97 million Euros.
Economic Burden and Health-Related Quality of Life Associated with Current Treatments for Anaemia in Patients with CKD not on Dialysis: A Systematic Review
PharmacoEconomics - open. 2019
BACKGROUND The cost and health-related quality of life (HRQoL) burden associated with treatments for anaemia of chronic kidney disease (CKD) is not well characterized among non-dialysis-dependent (NDD) patients. OBJECTIVE Our objective was to review the literature on costs and HRQoL associated with current treatments for anaemia of CKD among NDD patients. METHODS The Cochrane Library, MEDLINE, Embase, NHS EED, and NHS HTA databases were searched for original studies published in English between 1 January 2000 and 17 March 2017. The following inclusion criteria were applied: adult population; primary focus was anaemia of CKD; patients received iron supplementation, red blood cell transfusion, or erythropoiesis-stimulating agents (ESAs); and reported results on HRQoL and/or costs. Studies that included NDD patients, did not compare different treatments, and had relevant designs were retained. HRQoL and cost outcomes were summarized in a narrative synthesis. RESULTS In total, 16 studies met the inclusion criteria: six randomized controlled trials, four prospective single-arm trials, three retrospective studies, one prospective observational study, one simulation study, and one cross-sectional survey. All included ESAs. Treatment of anaemia (compared with no treatment) was associated with HRQoL improvements in five of six studies and lower costs in four of four studies. Treatment aiming for higher haemoglobin targets (compared with lower targets) resulted in modest HRQoL improvements, higher healthcare resource utilization (HRU), and higher costs. CONCLUSIONS In NDD patients, untreated anaemia of CKD leads to higher costs, higher HRU, and lower HRQoL compared with initiating anaemia treatment. Relative to aiming for lower haemoglobin targets with ESAs, higher targets conferred modest HRQoL improvements and were associated with higher HRU.
Cost-effectiveness of continuous erythropoietin receptor activator in anemia
Clinicoeconomics & Outcomes Research. 2014;6:319-30.
BACKGROUND Erythropoiesis-stimulating agents (ESAs) are the mainstay of anemia therapy. Continuous erythropoietin receptor activator (CERA) is a highly effective, long-acting ESA developed for once-monthly dosing. A multitude of clinical studies has evaluated the safety and efficiency of this treatment option for patients with renal anemia. In times of permanent financial pressure on health care systems, the cost-effectiveness of CERA should be of particular importance for payers and clinicians. OBJECTIVE To critically analyze, from the nephrologists' point of view, the published literature focusing on the cost-effectiveness of CERA for anemia treatment. METHODS The detailed literature search covered electronic databases including MEDLINE, PubMed, and Embase, as well as international conference abstract databases. RESULTS Peer-reviewed literature analyzing the definite cost-effectiveness of CERA is scarce, and most of the available data originate from conference abstracts. Identified data are restricted to the treatment of anemia due to chronic kidney disease. Although the majority of studies suggest a considerable cost advantage for CERA, the published literature cannot easily be compared. While time and motion studies clearly indicate that a switch to CERA could minimize health care staff time in dialysis units, the results of studies comparing direct costs are more ambivalent, potentially reflecting the differences between health care systems and variability between centers. CONCLUSION Analyzed data are predominantly insufficient; they miss clear evidence and have to thus be interpreted with great caution. In this day and age of financial restraints, results from well-designed, head-to-head studies with clearly defined endpoints have to prove whether CERA therapy can achieve cost savings without compromising anemia management.
What is the clinical effectiveness and cost- effectiveness of erythropoietin-stimulating agents for the treatment of patients with cancer-treatment induced anaemia? Insights from cumulative meta-analyses (CMA) and lessons for cost-effectiveness analyses
Value in Health. 2014;17((7)):A617.
Cost-utility analysis of deferiprone for the treatment of beta-thalassaemia patients with chronic iron overload: a UK perspective
BACKGROUND Patients with beta-thalassaemia major experience chronic iron overload due to regular blood transfusions. Chronic iron overload can be treated using iron-chelating therapies such as desferrioxamine (DFO), deferiprone (DFP) and deferasirox (DFX) monotherapy, or DFO-DFP combination therapy. OBJECTIVES This study evaluated the relative cost effectiveness of these regimens over a 5-year timeframe from a UK National Health Service (NHS) perspective, including personal and social services. METHODS A Markov model was constructed to evaluate the cost effectiveness of the treatment regimens over 5years. Based on published randomized controlled trial evidence, it was assumed that all four treatment regimens had a comparable effect on serum ferritin concentration (SFC) and liver iron concentration (LIC), and that DFP was more effective for reducing cardiac morbidity and mortality. Published utility scores for route of administration were used, with subcutaneously administered DFO assumed to incur a greater quality of life (QoL) burden than the oral chelators DFP and DFX. Healthcare resource use, drug costs (2010/2011 costs), and utilities associated with adverse events were also considered, with the effect of varying all parameters assessed in sensitivity analysis. Incremental costs and quality-adjusted life-years (QALYs) were calculated for each treatment, with cost effectiveness expressed as incremental cost per QALY. Assumptions that DFP conferred no cardiac morbidity, mortality, or morbidity and mortality benefit were also explored in scenario analysis. RESULTS DFP was the dominant strategy in all scenarios modelled, providing greater QALY gains at a lower cost. Sensitivity analysis showed that DFP dominated all other treatments unless the QoL burden associated with the route of administration was greater for DFP than for DFO, which is unlikely to be the case. DFP had >99% likelihood of being cost effective against all comparators at a willingness-to-pay threshold of 20,000 per QALY. CONCLUSIONS In this analysis, DFP appeared to be the most cost-effective treatment available for managing chronic iron overload in beta-thalassaemia patients. Use of DFP in these patients could therefore result in substantial cost savings.
Hemoglobin level at initiation of darbepoetin alfa: impact on need for transfusion and associated costs in chemotherapy-induced anemia treatment in Europe
Supportive Care in Cancer. 2013;21((2):):485-93.
PURPOSE Erythropoiesis-stimulating agents can reduce red blood cell transfusion rates in patients developing anemia while receiving chemotherapy. We investigated potential cost savings from reduced transfusion rates in patients starting darbepoetin alfa (DA) at higher versus lower hemoglobin (Hb) levels. METHODS Two systematic literature reviews were performed: transfusion outcomes in patients receiving DA stratified by baseline Hb level and costs of transfusion in Europe. Potential cost savings were calculated by multiplying the difference in transfusion rates between Hb levels by the midpoint of transfusion costs. RESULTS Despite differences in baseline characteristics, treatment duration and analysis technique, the clinical studies (n=8) showed that fewer transfusions were required when DA was initiated at higher versus lower Hb levels. The economic studies (n=9) showed that 1unit of transfusion ranged from 130 to 537 (2010-adjusted values). Cost savings from initiating DA at higher versus lower Hb levels were 503-2,226 (2units transfused) and 880-3,895 (3.5units) per ten patients. CONCLUSIONS Transfusion incidence increases with DA initiation at lower Hb levels. Potential cost savings depend on the number of units transfused and cost items included. DA initiation according to guidelines can reduce transfusions and potentially reduce transfusion-associated costs. Journal Article.
Economic evaluation of erythropoiesis-stimulating agents for anemia related to cancer
BACKGROUND Erythropoiesis-stimulating agents (ESA) administered to cancer patients with anemia reduce the need for blood transfusions and improve quality-of-life (QOL). Concerns about toxicity have led to more restrictive recommendations for ESA use; however, the incremental costs and benefits of such a strategy are unknown. METHODS The authors created a decision model to examine the costs and consequences of ESA use in patients with anemia and cancer from the perspective of the Canadian public healthcare system. Model inputs were informed by a recent systematic review. Extensive sensitivity analyses and scenario analysis rigorously assessed QOL benefits and more conservative ESA administration practices (initial hemoglobin [Hb] <10 g/dL, target Hb < or =12 g/dL, and chemotherapy induced anemia only). RESULTS Compared with supportive transfusions only, conventional ESA treatment was associated with an incremental cost per quality-adjusted life year (QALY) gained of $267,000 during a 15-week time frame. During a 1.3-year time horizon, ESA was associated with higher costs and worse clinical outcomes. In scenarios where multiple assumptions regarding QOL all favored ESA, the lowest incremental cost per QALY gained was $126,000. Analyses simulating the use of ESA in accordance with recently issued guidelines resulted in incremental cost per QALY gained of > $100,000 or ESA being dominated (greater costs with lower benefit) in the majority of the scenarios, although greater variability in the cost-utility ratio was present. CONCLUSIONS Use of ESA for anemia related to cancer is associated with incremental cost-effectiveness ratios that are not economically attractive, even when used in a conservative fashion recommended by current guidelines.
Cost effectiveness of deferasirox compared to desferrioxamine in the treatment of iron overload in lower-risk, transfusion-dependent myelodysplastic syndrome patients
Journal of Medical Economics. 2010;13((3):):559-70.
Objective: The study evaluated the cost effectiveness of deferasirox (Exjade) compared to non-proprietary desferrioxamine (DFO) for the control of transfusional iron overload in lower risk myelodysplastic syndromes (MDS) patients. A UK National Health Service perspective was adopted. Methods: Recent clinical evidence has demonstrated the efficacy and safety of deferasirox in transfusion-dependent MDS patients with elevated serum ferritin levels. An economic model was used to extrapolate the clinical benefits of iron chelation therapy (ICT) in a cohort of lower risk MDS patients. Costs for drug acquisition, drug administration and monitoring, and quality of life (utility) outcomes associated with mode of drug administration were derived from a variety of sources. The incremental cost per QALY gained for deferasirox was estimated. Costs and outcomes were discounted at 3.5% in line with UK standards. Results: The base-case cost effectiveness of deferasirox versus DFO was estimated to be 20,822 per QALY gained, the key driver being the additional quality of life benefits associated with a simpler mode of administration for deferasirox. A mean survival benefit for both forms of ICT of 4.5 years was estimated. The results were sensitive to drug dose, days of DFO administration, and patient weight. Conclusions: In the UK, a cost per QALY below 20,000-30,000 is considered cost effective. Hence, the results from this economic analysis suggest deferasirox is cost effective in lower risk, transfusion-dependent, MDS patients. Limitations with the analysis include a lack of comparative randomised controlled trial evidence, in particular to differentiate survival and clinical outcomes for deferasirox and DFO.
Probabilistic cost-minimisation analysis of darbepoetin alpha versus epoetin alpha in treating anaemia secondary to chronic renal failure. Assessment in Spanish clinical practice
Farmacia Hospitalaria. 2009;33((4):):208-16.
INTRODUCTION The direct transfer of the results of pharmaco-economic studies between countries may not be suitable if the proper adaptations are not made to take into account differences in treatment patterns, resource use and costs from country to country. OBJECTIVE To estimate the cost in Spain of treating anaemia secondary to chronic renal failure with darbepoetin alpha or epoetin alpha from a review and analysis of available current information. In addition, the role of the route of administration as a main driver of the cost will be analysed. METHOD population: patients with chronic kidney failure induced anaemia. Data: Medline and Embase search of studies directly comparing erythropoiesis stimulating agents. ANALYSIS Cost minimization analysis from the perspective of a hospital pharmacy department. The main outcome chosen was the difference between the average cost per patient undergoing a 30-day treatment with epoetin alpha versus darbepoetin alpha. RESULTS (a) haemodialysis: changing from epoetin alpha to darbepoetin alpha is associated with a cost reduction of 8.67%; CI 95%, -1.34 to 17.92 (euro 17.48; CI 95%, -2.70 to 36.13); probabilistic analysis showed that the use of darbepoetin alpha could be associated with a cost-saving probability of 94.9%. The IV administration yielded a decrease in costs of about 16.00%; CI 95%, -2.38 to 36.77 (euro 41.78, CI 95%: -6.21 to 96.04). (b) Pre-dialysis: darbepoetin alpha is associated with a cost reduction of about 11-32%. CONCLUSIONS The use of darbepoetin alpha for the treatment of chronic renal failure induced anaemia (haemodialysis and pre-dialysis) shows higher cost efficiency than epoetin alpha in Spain; these differences increase with IV administration.