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

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.

10.36469/9870 ◽  
2013 ◽  
Vol 1 (3) ◽  
pp. 239-253 ◽  
Author(s):  
Jona T. Stahmeyer ◽  
Svenja Schauer ◽  
Siegbert Rossol ◽  
Hans Heinrich Wedemeyer ◽  
Daniel Wirth ◽  
...  

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.


2019 ◽  
Vol 21 (Supplement_3) ◽  
pp. iii68-iii69
Author(s):  
X Armoiry ◽  
P Auguste ◽  
C Dussart ◽  
J Guyotat ◽  
M Connock

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


2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 557-557
Author(s):  
K. E. Ougari ◽  
C. Taneja ◽  
O. Sofrygin ◽  
S. Kaura ◽  
T. Delea

557 Background: The Austrian Breast and Colorectal Cancer Study Group Trial 12 (ABCSG-12) examined the efficacy of 3 years (yrs) of treatment with goserelin in combination with ET (anastrozole or tamoxifen) with or without ZOL 4 mg q6 mos in 1,803 premenopausal women with EBC (median age 45 yrs). After a median follow-up of 47.8 mos (max 84 mos), risk of disease-free survival (DFS) events was reduced by 36% (HR = 0.64; p = 0.01) in patients (pts) who received ZOL (ZOL+ET) compared with those who did not (ET). Methods: A Markov model was used to estimate the cost per quality adjusted life years (QALYs) gained of 3 years treatment duration of ZOL+ET versus ET-only in premenopausal women with EBC based on results of the ABCSG-12. A Canadian healthcare system perspective and a lifetime timeframe were used. Outcomes and cost of breast cancer recurrence were based on recent published studies. Results were generated under 2 scenarios regarding duration of benefit (reduction in risk of recurrence) with ZOL: (1) Benefits persist to maximum follow-up in ABCSG-12 (trial benefit); (2) Benefits persist until death (lifetime benefit). Results: The cost of 3 years of ZOL (medication and administration) is 4 191 $CDN. Under the lifetime benefit scenario, 73% of these costs are offset by savings in the cost of recurrences. Under the trial benefit scenario, 12% are offset. QALYs gained are 1.63 yrs and 0.52 yrs under the lifetime and trial benefit scenarios respectively; cost-effectiveness is 1 122 $CDN and 3 675 $CDN per QALY gained respectively, which is well below the 50 000 $CDN per QALY threshold frequently used to assess whether therapies are cost-effective. Conclusions: The combination of ZOL + ET is a cost-effective use of healthcare resources from a Canadian healthcare system perspective. [Table: see text]


2015 ◽  
Vol 36 (4) ◽  
pp. 438-444 ◽  
Author(s):  
Raghu U. Varier ◽  
Eman Biltaji ◽  
Kenneth J. Smith ◽  
Mark S. Roberts ◽  
M. Kyle Jensen ◽  
...  

OBJECTIVEClostridium difficile infection (CDI) places a high burden on the US healthcare system. Recurrent CDI (RCDI) occurs frequently. Recently proposed guidelines from the American College of Gastroenterology (ACG) and the American Gastroenterology Association (AGA) include fecal microbiota transplantation (FMT) as a therapeutic option for RCDI. The purpose of this study was to estimate the cost-effectiveness of FMT compared with vancomycin for the treatment of RCDI in adults, specifically following guidelines proposed by the ACG and AGA.DESIGNWe constructed a decision-analytic computer simulation using inputs from the published literature to compare the standard approach using tapered vancomycin to FMT for RCDI from the third-party payer perspective. Our effectiveness measure was quality-adjusted life years (QALYs). Because simulated patients were followed for 90 days, discounting was not necessary. One-way and probabilistic sensitivity analyses were performed.RESULTSBase-case analysis showed that FMT was less costly ($1,669 vs $3,788) and more effective (0.242 QALYs vs 0.235 QALYs) than vancomycin for RCDI. One-way sensitivity analyses showed that FMT was the dominant strategy (both less expensive and more effective) if cure rates for FMT and vancomycin were ≥70% and <91%, respectively, and if the cost of FMT was <$3,206. Probabilistic sensitivity analysis, varying all parameters simultaneously, showed that FMT was the dominant strategy over 10, 000 second-order Monte Carlo simulations.CONCLUSIONSOur results suggest that FMT may be a cost-saving intervention in managing RCDI. Implementation of FMT for RCDI may help decrease the economic burden to the healthcare system.Infect Control Hosp Epidemiol 2014;00(0): 1–7


2002 ◽  
Vol 146 (3) ◽  
pp. 283-294 ◽  
Author(s):  
GM Vidal-Trecan ◽  
JE Stahl ◽  
I Durand-Zaleski

OBJECTIVE: To examine the cost-effectiveness of therapeutic strategies for patients with toxic thyroid adenoma. DESIGN: A decision analytic model was used to examine strategies, including thyroid lobectomy after a 3-month course of antithyroid drugs (ATDs), radioactive iodine (RAI), and lifelong ATDs followed by either RAI (ATD-RAI) or surgery (ATD-surgery) in patients suffering severe drug reactions. METHODS: Outcomes were measured in quality-adjusted life years. Data on the prevalence of co-incident thyroid cancer, complications and treatment efficacies were derived from a systematic review of the literature (1966-2000). Costs were examined from the health care system perspective. Costs and effectiveness were examined at their present values. Discounting (3% per year), variations of major cost components, and every variable for which disagreements exist among studies or expert opinion were examined by sensitivity analyses. RESULTS: For a 40-year-old woman, surgery was both the most effective and the least costly strategy (Euro 1391),while ATD-RAI cost the most (Euro 5760). RAI was more effective than surgery if surgical mortality exceeded 0.6% (base-case 0.001%). RAI become less costly for women of more than 72 years (more than 66 in discounted analyses). For women of 85, ATD-RAI may be more effective than RAI and have an inexpensive marginal cost-effectiveness ratio (Euro 4975) if lifelong follow-up results in no decrement in quality of life. CONCLUSIONS: Age, surgical mortality, therapeutic costs and patient preference must all be considered in choosing an appropriate therapy.


2021 ◽  
Vol 9 ◽  
Author(s):  
Ting Zhou ◽  
Yanan Sheng ◽  
Haijing Guan ◽  
Rui Meng ◽  
Zijing Wang

Objective: To evaluate the cost effectiveness of vedolizumab vs. infliximab in the treatment of anti-tumor necrosis factor-alpha (TNF-α)-naïve patients with moderate-to-severe active ulcerative colitis (UC) in China.Methods: The costs and effectiveness of vedolizumab and infliximab in the treatment of anti-TNF-α naïve patients with moderate-to-severe active UC were compared using a hybrid decision tree model and a Markov model. From the perspective of the Chinese healthcare system, this study simulated the lifetime health benefits [quality-adjusted life-years (QALYs)] and costs (USD) for patients with UC from the induction phase to the maintenance phase, with an annual discount rate of 5%. The clinical efficacy and transition probability data were based on a previously published network meta-analysis. The health utility, surgical risk, biologic drug discontinuation rate, and mortality were derived from previous literature and the Chinese statistical yearbook. The cost data were based on China's drug purchase and biding platform and the results of a survey sent to clinicians in 18 tertiary hospitals. One-way and probabilistic sensitivity analyses (PSAs) were performed to validate the robustness of the models' assumptions and specific parameter estimates.Results: The results of the base-case analyses showed that compared with infliximab, vedolizumab led to a gain of 0.25 QALYs (9.56 vs. 9.31 QALYs) and was less expensive by $7,349 ($180,138 vs. 187,487), indicating that the use of vedolizumab was a dominant strategy. The results of one-way sensitivity analyses suggested that the annual discount rate and health-state costs had the greatest impact, but the results were otherwise consistent with those of the base-case analyses. The PSAs suggested that vedolizumab had a 98.6% probability of being effective at a threshold of 3 times the gross domestic product (GDP) per capita in China in 2020.Conclusion: Compared with infliximab, vedolizumab appears to be a more cost-effective option in the treatment of anti-TNF-α naïve adult patients with moderate-to-severe, active UC in China.


BMJ Open ◽  
2020 ◽  
Vol 10 (11) ◽  
pp. e040691
Author(s):  
Qiao Liu ◽  
Xia Luo ◽  
Liubao Peng ◽  
Lidan Yi ◽  
Xiaomin Wan ◽  
...  

ObjectiveTo investigate the cost-effectiveness of ramucirumab plus erlotinib compared with placebo plus erlotinib in the first-line setting for patients with EGFR-mutated metastatic non-small cell lung cancer (mNSCLC) from the Chinese healthcare system perspective.DesignA Markov model consisting of three health states using clinical survival data from the RELAY phase III randomised clinical trial, a lifetime horizon for costs and quality-adjusted life-years (QALYs) was constructed to analyse the cost-effectiveness of ramucirumab plus erlotinib. One-way and probabilistic sensitivity analyses were performed to evaluate the robustness of the model. Additional price reduction scenario analyses were performed.SettingThe Chinese healthcare system perspective.ParticipantsA hypothetical Chinese cohort of patients with confirmed previously documented ex19del or Leu858Arg mutation stage IV NSCLC, and without known epidermal growth factor receptor (EGFR) Thr790Met mutation and central nervous system metastases.InterventionsRamucirumab plus erlotinib versus placebo plus erlotinib.Primary outcome measureCosts, QALYs, incremental cost-effectiveness ratio (ICER).ResultsIn base-case analysis, ramucirumab plus erlotinib yield an additional 4.21 QALYs at a cost of $540 590, resulting in an ICER of $128 302/QALY. In price reduction scenario analysis, the ICER ($65 227/QALY) was decreased significantly when the National Reimbursement Drug List (NRDL) negotiation was available for ramucirumab, and the ICER ($131 554/QALY) was increased slightly when the NRDL negotiation was unavailable for erlotinib. Sensitivity analyses demonstrated our results to be most sensitive to the unit cost of ramucirumab (10 mg/kg), and more than 52.1% reduction in the price of ramucirumab resulted in the ICER under the willingness-to-pay threshold set for affluent regions ($70 353/QALY).ConclusionsRamucirumab plus erlotinib is unlikely to be cost-effective for patients with untreated EGFR-mutated mNSCLC in China. Reducing the price of ramucirumab through the National Healthcare Security Administration negotiation was found to be the most realistic action to improve cost-effectiveness.


2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e19529-e19529 ◽  
Author(s):  
Josh John Carlson ◽  
Benjamin Eckert ◽  
Marita Zimmerman

e19529 Background: MRD testing is part of the response evaluation criteria for MM, and clinical guidelines recommend MRD testing with technologies that detect malignant cells at a level of at least 1/100,000. Deep MRD negativity correlates with improved health outcomes for MM pts, and MRD status may inform treatment (tx) continuation decisions during MM maintenance therapy. We evaluated the potential cost-effectiveness of NGS MRD (clonoSEQ) vs. no MRD testing in the maintenance MM setting. Methods: We developed a Markov model with 6 health states: MRD+ or MRD- on or off tx, relapsed, or dead and compared yearly NGS MRD to no MRD testing over a lifetime horizon. The model assumed tx stops after an MRD- result; otherwise, tx continued until disease progression. Progression-free survival (PFS) and overall survival (OS) data for MRD- and MRD+ and hazard ratios for on/off tx were applied based on peer-reviewed literature. MRD- pts were assumed to have the same PFS/OS rates on and off tx, which were varied in sensitivity analyses. On tx, 2%/month of MRD+ pts became MRD-. Health state utilities were based on peer-reviewed literature and included an adverse event (AE) disutility. The cost of NGS MRD was $1,800 + $1,836 for specimen collection, with maintenance tx at $21,168/month and tx for relapsed pts at $27,422/month. We used a US health system perspective and a 3% discount rate. Results: MRD testing saved $1,156,600 over patients remaining lifetime. Health outcomes were similar and slightly favored MRD testing vs no testing (0.01 quality-adjusted life-years [QALYs]) from lower AE risk over time, suggesting a potentially dominant strategy, i.e., less costly with improved health outcomes. This result was most sensitive to the probability of MRD+ to MRD- transition and the cost of maintenance tx. Conclusions: NGS MRD testing is cost-saving, with potential QALY gains due to avoidance of tx-related AEs compared with no testing for MM patients on maintenance therapy. Further studies are needed to determine the health outcomes of MRD testing during MM maintenance tx. [Table: see text]


2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Li-Jen Cheng ◽  
Swee Sung Soon ◽  
Teck Wei Tan ◽  
Cher Heng Tan ◽  
Terence Sey Kiat Lim ◽  
...  

Abstract Background To evaluate the cost-effectiveness of six diagnostic strategies involving magnetic resonance imaging (MRI) targeted biopsy for diagnosing prostate cancer in initial and repeat biopsy settings from the Singapore healthcare system perspective. Methods A combined decision tree and Markov model was developed. The starting model population was men with mean age of 65 years referred for a first prostate biopsy due to clinical suspicion of prostate cancer. The six diagnostic strategies were selected for their relevance to local clinical practice. They comprised MRI targeted biopsy following a positive pre-biopsy multiparametric MRI (mpMRI) [Prostate Imaging – Reporting and Data System (PI-RADS) score ≥ 3], systematic biopsy, or saturation biopsy employed in different testing combinations and sequences. Deterministic base case analyses with sensitivity analyses were performed using costs from the healthcare system perspective and quality-adjusted life years (QALY) gained as the outcome measure to yield incremental cost-effectiveness ratios (ICERs). Results Deterministic base case analyses showed that Strategy 1 (MRI targeted biopsy alone), Strategy 2 (MRI targeted biopsy ➔ systematic biopsy), and Strategy 4 (MRI targeted biopsy ➔ systematic biopsy ➔ saturation biopsy) were cost-effective options at a willingness-to-pay (WTP) threshold of US$20,000, with ICERs ranging from US$18,975 to US$19,458. Strategies involving MRI targeted biopsy in the repeat biopsy setting were dominated. Sensitivity analyses found the ICERs were affected mostly by changes to the annual discounting rate and prevalence of prostate cancer in men referred for first biopsy, ranging between US$15,755 to US$23,022. Probabilistic sensitivity analyses confirmed Strategy 1 to be the least costly, and Strategies 2 and 4 being the preferred strategies when WTP thresholds were US$20,000 and US$30,000, respectively. Limitations and conclusions This study found MRI targeted biopsy to be cost-effective in diagnosing prostate cancer in the biopsy-naïve setting in Singapore.


2021 ◽  
Author(s):  
Jefferson Antonio Buendia ◽  
Diana Guerrero Patino

Abstract BackgroundAn important proportion of asthma patients remain uncontrolled despite the use of inhaled corticosteroids and long-acting beta-agonists. Some add-on therapies, as tiotropium bromide have been recommended for this subgroup of patients. The purpose of this study was to assess the cost-effectiveness of tiotropium as add-on therapies to ICS + LABA for patients with severe asthma. Methods A probabilistic Markov model was created to estimate the cost and quality-adjusted life-years (QALYs) of patients with severe asthma in Colombia. Total costs and QALYS of two interventions including standard therapy (ICS + LABA), add-on therapy with tiotropium, were calculated over a lifetime horizon. Multiple sensitivity analyses were conducted. Cost-effectiveness was evaluated at a willingness-to-pay value of $19,000. ResultsThe model suggests a potential gain of 1.06 QALYs per patient per year on tiotropium, with a difference of US$ 478 in favor of tiotropium; showing dominance respect to standard therapy. A position of dominance negates the need to calculate an incremental cost‐effectiveness ratio. In the deterministic sensitivity analyses, our base‐case results were robust to variations of all assumptions and parameters Conclusion Add-on therapy with tiotropium was found to be cost-effective when added to usual care in patients who remain uncontrolled despite treatment with medium or high-dose ICS/LABA. Our study provides evidence that should be used by decision-makers to improve clinical practice guidelines and should be replicated to validate their results in other middle-income countries.


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