scholarly journals Cost-Effectiveness Analysis for the Treatment of Hyperphosphatemia in Predialysis Patients: Calcium-Based versus Noncalcium-Based Phosphate Binders

2018 ◽  
Vol 2018 ◽  
pp. 1-7
Author(s):  
B. L. Goh ◽  
A. Soraya ◽  
A. Goh ◽  
K. L. Ang
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Sensitivity Analyses ◽  
Phosphate Binders ◽  
Base Case ◽  
Health State ◽  
Life Years ◽  
High Calcium ◽  
Calcium Load ◽  
Markov Decision Model

Background. Hyperphosphatemia in chronic kidney disease (CKD) patients is often treated with calcium carbonate (CaCO3) despite the fact that CaCO3 is associated with increased calcium load and potentially increased cardiovascular risk. Alternative treatments with noncalcium-based phosphate binders do not increase the calcium load but are more costly. This study analyzes the cost-effectiveness of sevelamer versus CaCO3 for the treatment of hyperphosphatemia in stage III-V predialysis CKD patients in Malaysia. Methods. A Markov decision model was adapted to simulate a hypothetical cohort of CKD patients requiring treatment for hyperphosphatemia. Survival was estimated by using efficacy data from the INDEPENDENT-CKD clinical trial. Cost data was obtained from Malaysian studies while health state utilities were derived from literature. Analysis was performed over lifetime duration from the perspective of the Ministry of Health Malaysia with 2013 as reference year. Results. In the base case analysis, sevelamer treatment gained 6.37 life years (5.27 QALY) compared to 4.25 life years (3.54 QALY) with CaCO3. At 3% discount, lifetime costs were RM159,901 ($48,750) and RM77,139 ($23,518) on sevelamer and CaCO3, respectively. Incremental cost-effectiveness (ICER) of sevelamer versus CaCO3 was RM47,679 ($14,536) per QALY, which is less than the WHO threshold of three times GDP per capita (RM99,395) per QALY. Sensitivity analyses, both using scenario sensitivity analysis and probabilistic sensitivity analysis, showed the result to be robust. Conclusions. Our study finds that sevelamer is potentially cost-effective compared to CaCO3, for the treatment of hyperphosphatemia in predialysis CKD III-V. We propose that sevelamer should be an option in the treatment of Malaysian predialysis patients with hyperphosphatemia, particularly those with high calcium load.

Cost-effectiveness of mobile health-based integrated care for atrial fibrillation: Model development and data analysis (Preprint)

2021 ◽  
Author(s):  
Xueyan Luo ◽  
Wei Xu ◽  
Quan Yuan ◽  
Han Lai ◽  
Chunji Huang
Keyword(s):  
Atrial Fibrillation ◽  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Integrated Care ◽  
Mobile Health ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Life Years ◽  
Mhealth Technology

BACKGROUND Mobile health (mhealth) technology is increasingly used in disease management. Using mhealth tools to integrate and streamline care was found to improve atrial fibrillation (AF) patients’ clinical outcomes. OBJECTIVE This study aimed to investigate the potential clinical and health economic outcomes of mhealth-based integrated care for AF from the perspective of a public healthcare provider in China. METHODS A Markov model was designed to compare outcomes of mhealth-based care and usual care in a hypothetical cohort of AF patients in China. The time horizon was 30 years with monthly cycles. Model outcomes measured were direct medical cost, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). Sensitivity analyses were conducted to examine the robustness of base-case results. RESULTS In the base-case analysis, mhealth-based care gained higher QALYs of 0.0818 with an incurred cost of USD1,778. Using USD33,438 per QALY (three times gross domestic product) as the willingness-to-pay threshold, mhealth-based care was cost-effective, with an ICER of USD21,739 per QALY. The one-way sensitivity analysis found compliance to mhealth-based care had the greatest impact on the ICER. In probabilistic sensitivity analysis, mhealth-based care was accepted as cost-effective in 80.91% of 10,000 iterations. CONCLUSIONS This study suggested that the use of mhealth technology in streamlining and integrating care for AF patients was cost-effective in China.

scholarly journals Cost-Effectiveness Analysis of 68Ga DOTA-TATE PET/CT, 111In-Pentetreotide SPECT/CT and CT for Diagnostic Workup of Neuroendocrine Tumors

Diagnostics ◽  
2021 ◽  
Vol 11 (2) ◽  
pp. 334
Author(s):  
Matthias Frank Froelich ◽  
Moritz Ludwig Schnitzer ◽  
Adrien Holzgreve ◽  
Felix Gerhard Gassert ◽  
Eva Gresser ◽  
...  
Keyword(s):  
Computed Tomography ◽  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Neuroendocrine Tumors ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Emission Computed Tomography ◽  
Life Years ◽  
Pet Ct ◽  
Therapy Costs

Neuroendocrine tumors (NETs) are relatively rare neoplasms arising from the hormone-producing neuroendocrine system that can occur in various organs such as pancreas, small bowel, stomach and lung. As the majority of these tumors express somatostatin receptors (SSR) on their cell membrane, utilization of SSR analogs in nuclear medicine is a promising, but relatively costly approach for detection and localization. The aim of this study was to analyze the cost-effectiveness of 68Ga-DOTA-TATE PET/CT (Gallium-68 DOTA-TATE Positron emission tomography/computed tomography) compared to 111In-pentetreotide SPECT/CT (Indium-111 pentetreotide Single Photon emission computed tomography/computed tomography) and to CT (computed tomography) alone in detection of NETs. A decision model on the basis of Markov simulations evaluated lifetime costs and quality-adjusted life years (QALYs) related to either a CT, SPECT/CT or PET/CT. Model input parameters were obtained from publicized research projects. The analysis is grounded on the US healthcare system. Deterministic sensitivity analysis of diagnostic parameters and probabilistic sensitivity analysis predicated on a Monte Carlo simulation with 30,000 reiterations was executed. The willingness-to-pay (WTP) was determined to be $ 100,000/QALY. In the base-case investigation, PET/CT ended up with total costs of $88,003.07 with an efficacy of 4.179, whereas CT ended up with total costs of $88,894.71 with an efficacy of 4.165. SPECT/CT ended up with total costs of $89,973.34 with an efficacy of 4.158. Therefore, the strategies CT and SPECT/CT were dominated by PET/CT in the base-case scenario. In the sensitivity analyses, PET/CT remained a cost-effective strategy. This result was due to reduced therapy costs of timely detection. The additional costs of 68Ga-DOTA-TATE PET/CT when compared to CT alone are justified in the light of potential savings in therapy costs and better outcomes.

Cost-effectiveness of FOLFIRI + cetuximab vs FOLFIRI + bevacizumab in the first-line (1L) treatment of RAS wild-type (wt) metastatic colorectal cancer (mCRC) in Germany: Data from the FIRE-3 (AIO KRK-0306) study.

2018 ◽  
Vol 36 (4_suppl) ◽  
pp. 800-800 ◽  
Author(s):  
Sebastian Stintzing ◽  
Ilse van Oostrum ◽  
Chris Pescott ◽  
Alma Katharina Steinbach-Buechert ◽  
Bart Heeg ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Cost Utility ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Health State ◽  
Primary Tumors ◽  
Payer Perspective ◽  
Life Years ◽  
The Cost

800 Background: The randomized, phase 3 FIRE-3 trial evaluated 1L FOLFIRI + cetuximab or bevacizumab in patients with RAS wt mCRC; overall survival favored FOLFIRI + cetuximab by > 8 months. The purpose of this analysis was to evaluate the cost-effectiveness of FOLFIRI + cetuximab vs that of FOLFIRI + bevacizumab as 1L treatment for patients in Germany with RAS wt mCRC (including the patient subgroup with RAS wt, left-sided [LS] primary tumors, as LS is a predictive factor). Methods: A standard oncology 3–health-state partitioned survival cost-utility model was developed to analyze the costs and health benefits of FOLFIRI + cetuximab vs those of FOLFIRI + bevacizumab from a German payer perspective based on data from FIRE-3 and the literature. Health outcomes were reported in life-years (LYs) and quality-adjusted life-years (QALYs) gained. A 3.5% discounting rate was applied to the modeled costs and outcomes. Results: Discounted costs, health gains, and incremental cost-effectiveness ratios (ICERs) for patients with RAS wt (base case) and patients with RAS wt, LS (subgroup) mCRC are summarized in the Table. Probabilistic sensitivity analyses showed that at relevant European willingness-to-pay (WTP) thresholds of €55,000 and €80,000, FOLFIRI + cetuximab had a 64.0% and 81.6% (base case) and 80.5% and 92.4% (subgroup) probability of being cost-effective vs FOLFIRI + bevacizumab, respectively. Clinical trial information: NCT00433927. Conclusions: Based on our analyses, FOLFIRI + cetuximab is cost-effective compared with FOLFIRI + bevacizumab in patients in Germany with RAS wt mCRC at official WTP thresholds applied by relevant European health technology assessment agencies. The cost-effectiveness of FOLFIRI + cetuximab is more pronounced in the subgroup of patients with RAS wt, LS tumors.[Table: see text]

Cost-effectiveness of atezolizumab monotherapy versus pembrolizumab monotherapy for first-line (1L) treatment of metastatic non-small cell lung cancer (mNSCLC).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e18847-e18847
Author(s):  
Chia-Wei Lin ◽  
Katherine Rosettie ◽  
Pinar Bilir ◽  
Hazal Celik ◽  
Seye Abogunrin ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
First Line ◽  
Deterministic Sensitivity Analysis ◽  
Anaplastic Lymphoma ◽  
Life Years

e18847 Background: Atezolizumab monotherapy is indicated as 1L treatment for mNSCLC patients with high programmed death ligand-1 (PD-L1) expression (≥ 50%) and without epidermal growth factor receptor or anaplastic lymphoma kinase mutations. This analysis assessed the cost-effectiveness of 1L atezolizumab monotherapy vs. pembrolizumab monotherapy for mNSCLC patients with high PD-L1 expression from a US third-party payer perspective. Methods: A Markov model with progression-free, progressive disease (PD), and death states was developed in Microsoft Excel to compare clinical and cost outcomes of atezolizumab monotherapy vs. pembrolizumab monotherapy. Efficacy, safety, and utility data were derived from systematic reviews and indirect comparisons of the IMpower110, Keynote-024, and Keynote-042 trials. Product prescribing information and clinical trials informed dosing and administration. Wholesale acquisition cost (WAC, accessed in January 2021) for drugs were used while other cost inputs were derived from publicly available fee schedules and peer-reviewed literature. The key outcome of interest was the incremental cost-effectiveness ratio (ICER) expressed as cost per quality-adjusted life-year (QALY) gained. Deterministic sensitivity analysis with 20% variation and probabilistic sensitivity analyses (PSA) were performed to address uncertainties around input parameters. Results: In the base case, 1L atezolizumab monotherapy was projected to increase life expectancy for patients by 0.60 life-years (4.35 vs. 3.75) and 0.47 QALYs (3.46 vs. 2.98) over pembrolizumab monotherapy at an incremental cost of $27,947 (mean total cost: $396,811 vs. $368,864), resulting in an ICER of $58,841/QALY gained. Results of the deterministic sensitivity analysis were most sensitive to changes in discount rates for costs and care costs in the PD state. The PSA showed that the probability of atezolizumab being cost-effective at willingness-to-pay thresholds of $100,000 and $150,000 was 41% and 49%, respectively. Conclusions: First-line atezolizumab monotherapy had 0.6 life-years and 0.47 QALYs gained compared with pembrolizumab monotherapy and was estimated to be cost-effective (ICER $58,841/QALY). As the ICER falls below the US cost-effectiveness thresholds ( < $100,000-$150,000/QALY), clinicians and payers should consider atezolizumab monotherapy as a cost-effective 1L option for mNSCLC patients with high PD-L1 expression.

COST-EFFECTIVENESS ANALYSIS OF IVABRADINE IN TREATMENT OF PATIENTS WITH HEART FAILURE IN IRAN

2018 ◽  
Vol 34 (6) ◽  
pp. 576-583 ◽  
Author(s):  
Saeed Taheri ◽  
Elham Heidari ◽  
Mohammad Ali Aivazi ◽  
Mehran Shams-Beyranvand ◽  
Mehdi Varmaghani
Keyword(s):  
Heart Failure ◽  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Transition Probabilities ◽  
Drug Acquisition ◽  
Sensitivity Analyses ◽  
Baseline Heart Rate ◽  
Life Years ◽  
The Cost

Objectives:This study aimed to assess the cost-effectiveness of ivabradine plus standard of care (SoC) in comparison with current SoC alone from the Iranian payer perspective.Methods:A cohort-based Markov model was developed to assess the incremental cost-effectiveness ratio (ICER) over a 10-year time horizon in a cohort of 1,000 patients. The baseline transition probabilities between New York Heart Association (NYHA), mortality rate, and hospitalization rate were extracted from the literature. The effect of ivabradine on mortality, hospitalization, and NYHA improvement or worsening were retrieved from the SHIFT study. The effectiveness was measured as quality-adjusted life-years (QALYs) using the utility values derived from Iranian Heart Failure Quality of Life study. Direct medical costs were obtained from hospital records and national tariffs. Deterministic and probabilistic sensitivity analyses were conducted to show the robustness of the model.Results:Ivabradine therapy was associated with an incremental cost per QALY of USD $5,437 (incremental cost of USD $2,207 and QALYs gained 0.41) versus SoC. The probabilistic sensitivity analysis showed that ivabradine is expected to have a 60 percent chance of being cost-effective accepting a threshold of USD $6,550 per QALY. Furthermore, deterministic sensitivity analysis indicated that the model is sensitive to the ivabradine drug acquisition cost.Conclusions:The cost-effectiveness model suggested that the addition of ivabradine to SoC therapy was associated with improved clinical outcomes along with increased costs. The analysis indicates that the clinical benefit of ivabradine can be achieved at a reasonable cost in eligible heart failure patients with sinus rhythm and a baseline heart rate ≥ 75 beats per minute (bpm).

scholarly journals Cost-effectiveness of Triple Therapy with Telaprevir for Chronic Hepatitis C Virus Patients in Germany

10.36469/9870 ◽  
2013 ◽  
Vol 1 (3) ◽  
pp. 239-253 ◽  
Author(s):  
Jona T. Stahmeyer ◽  
Svenja Schauer ◽  
Siegbert Rossol ◽  
Hans Heinrich Wedemeyer ◽  
Daniel Wirth ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Hepatitis C ◽  
Triple Therapy ◽  
Sensitivity Analyses ◽  
Healthcare Sector ◽  
Base Case ◽  
Life Years ◽  
Previously Treated Patients ◽  
Previously Treated ◽  
The Cost

Background: About 400,000-500,000 people are infected with hepatitis C in Germany. Long-term consequences are the development of liver cirrhosis and hepatocellular carcinoma. The introduction of first generation protease inhibitors has significantly improved the treatment of hepatitis C genotype 1 patients. The aim of the study was to assess the cost-effectiveness of triple therapy with telaprevir in Germany. Methods: We used a Markov model on disease progression and natural history to assess the cost-effectiveness of triple therapy with telaprevir compared to standard treatment with pegylated interferon and ribavirin. Model structure and inputs were discussed with clinical experts. Deterministic and probabilistic sensitivity analyses were performed to verify the robustness of results. Results: The base-case analyses shows that triple therapy results in higher costs (untreated patients: €48,446 vs. €30,691; previously treated patients: €63,228 vs. €48,603) and better outcomes (untreated patients: 16.85 qualily of life years [QALYs] vs. 15.97 QALYs; previously treated patients: 14.16 QALYs vs. 12.89 QALYs). The incremental cost-effectiveness ratio (ICER) was €20,131 per QALY and €30,567 per life year gained (LYG) for previously untreated patients. ICER in treatment experienced patients was €7,664 per QALY for relapse patients, €12,506 per QALY for partial responders and €28,429 per QALY for null responders. Results were robust in sensitivity analyses. Conclusion: Although triple therapy with telaprevir leads to additional costs, there is a high probability of being cost-effective for different thresholds. This health economic analysis makes an important contribution to current debates on cost savings and efficient resource allocation in the German healthcare sector.

scholarly journals Milk Powder Fortified with Potassium and Phytosterols to Decrease the Risk of Cardiovascular Events among the Adult Population in Malaysia: A Cost-Effectiveness Analysis

Nutrients ◽  
2019 ◽  
Vol 11 (6) ◽  
pp. 1235
Author(s):  
Anita E. Gandola ◽  
Livia Dainelli ◽  
Diane Zimmermann ◽  
Maznah Dahlui ◽  
Patrick Detzel
Keyword(s):  
Cost Effectiveness ◽  
Adult Population ◽  
Grey Literature ◽  
Milk Powder ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Discounted Cost ◽  
Life Years ◽  
The Impact

This study evaluated the cost-effectiveness of the consumption of a milk powder product fortified with potassium (+1050.28 mg/day) and phytosterols (+1200 mg/day) to lower systolic blood pressure and low-density lipoprotein cholesterol, respectively, and, therefore, the risk of myocardial infarction (MI) and stroke among the 35–75-year-old population in Malaysia. A Markov model was created against a do-nothing option, from a governmental perspective, and with a time horizon of 40 years. Different data sources, encompassing clinical studies, practice guidelines, grey literature, and statistical yearbooks, were used. Sensitivity analyses were performed to evaluate the impact of uncertainty on the base case estimates. With an incremental cost-effectiveness ratio equal to international dollars (int$) 22,518.03 per quality-adjusted life-years gained, the intervention can be classified as very cost-effective. If adopted nationwide, it would help prevent at least 13,400 MIs, 30,500 strokes, and more than 10,600 and 17,100 MI- and stroke-related deaths. The discounted cost savings generated for the health care system by those who consume the fortified milk powder would amount to int$8.1 per person, corresponding to 0.7% of the total yearly health expenditure per capita. Sensitivity analyses confirmed the robustness of the results. Together with other preventive interventions, the consumption of milk powder fortified with potassium and phytosterols represents a cost-effective strategy to attenuate the rapid increase in cardiovascular burden in Malaysia.

scholarly journals P14.12 The cost-effectiveness of tumor-treating fields in patients with newly diagnosed glioblastoma

2019 ◽  
Vol 21 (Supplement_3) ◽  
pp. iii68-iii69
Author(s):  
X Armoiry ◽  
P Auguste ◽  
C Dussart ◽  
J Guyotat ◽  
M Connock
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Survival Model ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Tumor Treating Fields ◽  
Kaplan Meier ◽  
Life Years ◽  
Secondary Analyses ◽  
The Cost

Abstract BACKGROUND The addition of novel therapy “Tumor-Treating fields” (TTF) to standard radio-chemotherapy with Temozolomide (TMZ) has recently shown superiority over conventional TMZ regimen in patients with glioblastoma. Despite the clinical benefit of TTF, there is a strong concern regarding the cost of this new treatment. A first cost-effectiveness analysis, which was published in 2016, was based on effectiveness outcomes from an interim analysis of the pivotal trial and used a “standard” Markov model. Here, we aimed to update the cost-effectiveness evaluation using a partitioned survival model design and using the latest effectiveness data. MATERIAL AND METHODS A partitioned survival model was developed with three mutually exclusive health states: stable disease, progressive disease, and dead. Parametric models were fitted to the Kaplan-Meier data for overall and progression-free survival. These generated clinically plausible extrapolations beyond the observed data. The perspective of the French national health insurance was adopted and the time horizon was 20 years. Base case results were expressed as cost/life-years (LY) gained (LYG). Secondary analyses were undertaken, with the results presented as cost/per quality adjusted life years (QALY) gained. Last, we undertook deterministic and probabilistic sensitivity analyses. RESULTS After applying 4% annual discounting of benefits and costs, the base case model generated incremental benefit of 0.507 LY at a incremental cost of €258,695 yielding an incremental cost effectiveness ratio (ICER) of €510,273 / LYG. Secondary analyses yielded an ICER of €667,173/QALY. Sensitivity analyses and bootstrapping methods showed the model was relatively robust. The model was sensitive to TTF device costs and the parametric model fitted to the Kaplan-Meier data for overall survival. The cost-effectiveness acceptability curve showed TTF has 0% of being cost-effective under conventional thresholds. CONCLUSION Using a partitioned survival model, uprated costs and more mature survival outcomes, TTF when compared to standard radio-chemotherapy with TMZ is not likely to be cost-effective. This has major implications in terms of access of newly eligible patients

scholarly journals Cost-effectiveness analysis of tranexamic acid for the treatment of traumatic brain injury, based on the results of the CRASH-3 randomised trial: a decision modelling approach

2020 ◽  
Vol 5 (9) ◽  
pp. e002716
Author(s):  
Jack Williams ◽  
Ian Roberts ◽  
Haleema Shakur-Still ◽  
Fiona E Lecky ◽  
Rizwana Chaudhri ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Tranexamic Acid ◽  
Randomised Trial ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Risk Of Death ◽  
Life Years ◽  
Markov Decision Model ◽  
The Uk ◽  
The Cost

IntroductionAn estimated 69 million traumatic brain injuries (TBI) occur each year worldwide, with most in low-income and middle-income countries. The CRASH-3 randomised trial found that intravenous administration of tranexamic acid within 3 hours of injury reduces head injury deaths in patients sustaining a mild or moderate TBI. We examined the cost-effectiveness of tranexamic acid treatment for TBI.MethodsA Markov decision model was developed to assess the cost-effectiveness of treatment with and without tranexamic acid, in addition to current practice. We modelled the decision in the UK and Pakistan from a health service perspective, over a lifetime time horizon. We used data from the CRASH-3 trial for the risk of death during the trial period (28 days) and patient quality of life, and data from the literature to estimate costs and long-term outcomes post-TBI. We present outcomes as quality-adjusted life years (QALYs) and 2018 costs in pounds for the UK, and US dollars for Pakistan. Incremental cost-effectiveness ratios (ICER) per QALY gained were estimated, and compared with country specific cost-effective thresholds. Deterministic and probabilistic sensitivity analyses were also performed.ResultsTranexamic acid was highly cost-effective for patients with mild TBI and intracranial bleeding or patients with moderate TBI, at £4288 per QALY in the UK, and US$24 per QALY in Pakistan. Tranexamic acid was 99% and 98% cost-effective at the cost-effectiveness thresholds for the UK and Pakistan, respectively, and remained cost-effective across all deterministic sensitivity analyses. Tranexamic acid was even more cost-effective with earlier treatment administration. The cost-effectiveness for those with severe TBI was uncertain.ConclusionEarly administration of tranexamic acid is highly cost-effective for patients with mild or moderate TBI in the UK and Pakistan, relative to the cost-effectiveness thresholds used. The estimated ICERs suggest treatment is likely to be cost-effective across all income settings globally.

Cost-Effectiveness of Deferasirox (Exjade®) Versus No Chelation In Patients with Lower Risk Myelodysplastic Syndromes (MDS): A Canadian Perspective

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4962-4962
Author(s):  
Khalid El Ouagari ◽  
Kristen Migliaccio-Walle ◽  
Helen Lau ◽  
Duygu Bozkaya
Keyword(s):  
Cost Effectiveness ◽  
Lower Risk ◽  
Chelation Therapy ◽  
Sensitivity Analyses ◽  
Base Case ◽  
System Perspective ◽  
Healthcare System Perspective ◽  
Life Years ◽  
The Cost ◽  
Canadian Healthcare

Abstract Abstract 4962 Introduction: Guidelines for the treatment of MDS recommend iron chelation therapy (ICT) in iron-overloaded lower-risk patients with MDS and candidates for stem cell transplantation. In particular, recent reports indicate that ICT may improve overall survival (OS) in transfusion-dependent patients with low or intermediate-1 (int-1) MDS as per international prognostic scoring system (IPSS) criteria. Deferasirox is a once-daily oral chelator, with easy administration and potentially better compliance. The goal of this study is to evaluate the cost-effectiveness of deferasirox compared to receiving no chelation therapy in transfusion-dependent patients with lower-risk MDS from a Canadian healthcare system perspective. Methods: A Markov model was developed to evaluate the cost-effectiveness of deferasirox compared to receiving no chelation therapy in transfusion-dependent patients with lower-risk (eg, IPSS low or int-1) MDS. The data used in the model were obtained from published or presented studies. Model outcomes, including life years (LY) gained, quality-adjusted life years (QALYs) gained, developing complications of iron overload, progressing to acute myeloid leukemia (AML), death, and direct medical costs of ICT, transfusion, complications and AML, were estimated for each treatment group based on a simulation of 1000 patient lives. Finally, incremental cost-effectiveness ratios (ICER) were calculated as the ratio of total medical costs to LY and QALY gains. Extensive one-way sensitivity analyses were performed to examine the effects of changes in key model parameters. Probabilistic sensitivity analyses were also performed. The outcomes of the model were evaluated over a 20-year time frame and discounted annually at the rate of 5%. Costs are reported in 2009 Canadian dollars (CAD$). Results: Under base case assumptions, patients receiving deferasirox were less likely to progress to cardiac disease, AML, and death compared to patients receiving no chelation therapy. Adding deferasirox was projected to increase OS by 4.46 years (undiscounted); discounting for time, OS was projected to be increased by 2.93 years. Furthermore, undiscounted QALYs were increased by 4.20 years and discounted QALYs, by 2.99 years. The clinical benefits of deferasirox are obtained at an additional expected discounted total lifetime cost of CAD$185,429. The incremental cost-effectiveness ratios were therefore estimated to be CAD$62,001/QALY gained and CAD$63,286/LY saved. Deterministic sensitivity analyses showed the base case results to be robust with respect to variations in assumptions and estimates. The cost-effectiveness acceptability curve shows that deferasirox was preferred to no treatment in 96% of simulations when the willingness to pay for a QALY was CAD$100,000. Conclusion: The results of our analysis indicate that deferasirox offers a cost-effective treatment option for patients with lower-risk MDS as the ICER is within the thresholds that are considered acceptable (ie, $50,000 to $100,000 per QALY gained), from a Canadian healthcare system perspective. Additional clinical studies are ongoing to evaluate event-free survival with deferasirox in patients with lower-risk MDS and transfusional iron overload. Disclosures: El Ouagari: Novartis: Employment. Migliaccio-Walle: Novartis: Research Funding. Lau: Novartis: Employment. Bozkaya: Novartis: Research Funding.

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