scholarly journals Cost–utility and budget impact analysis of tocilizumab for the treatment of refractory systemic juvenile idiopathic arthritis in Thailand

BMJ Open ◽  
2020 ◽  
Vol 10 (9) ◽  
pp. e037588
Author(s):  
Nitichen Kittiratchakool ◽  
Disorn Kulpokin ◽  
Chonticha Chanjam ◽  
Soamarat Vilaiyuk ◽  
Sirirat Charuvanij ◽  
...  

ObjectivesThis study aimed to analyse the cost–utility and budget impact of adding tocilizumab to the standard treatment for patients with refractory systemic juvenile idiopathic arthritis (sJIA) in Thailand.DesignEconomic evaluation using a decision-analytical model.SettingThailand.ParticipantsPatients with refractory sJIA who were ≥2 years old.MethodsThe use of tocilizumab as an add-on therapy to standard treatment was compared with standard treatment alone. A simulated health state transition model was used to estimate the lifetime costs and health outcomes from a societal perspective. Direct medical costs were collected from tertiary hospital databases while direct non-medical costs were derived from interviews. Health-related quality of life (QoL) was measured using the proxy version of three-level EuroQol five-dimensional questionnaire (EQ-5D-3L). Future costs and outcomes were discounted at an annual rate of 3%. The base case population was patients aged 9.41 years old at refractory disease onset. The results were reported as incremental cost-effectiveness ratios (ICER) in US dollar (USD). One-way and probabilistic sensitivity analysis were conducted to investigate parameter uncertainty. The 5-year budget impact was estimated from a governmental perspective.ResultsThe ICER of standard treatment plus tocilizumab was US$35 799 per quality-adjusted life-year (QALY) gained compared with standard treatment alone, which was not cost-effective at the threshold of US$5128 per QALY gained. The estimated 5 years budget impact was approximately US$4.8 million.ConclusionsThe use of standard treatment plus tocilizumab was not cost-effective in the Thai context, which has limited data. However, there is currently no second-line treatment for refractory sJIA in the Thai National List of Essential Medicines; thus, patients must receive higher doses of standard treatment which can cause many side effects. In contrast, tocilizumab showed obvious efficacy in clinical trials in improving treatment response and QoL. Therefore, the price of tocilizumab should be negotiated to reduce the financial impact on the healthcare system.

2020 ◽  
Author(s):  
Frederick Thielen ◽  
Marie-José Kersten ◽  
Pim Kuizenga ◽  
Mels Hoogendoorn ◽  
Eduardus FM Posthuma ◽  
...  

Abstract Background: Efficacy of lenalidomide plus rituximab (R-LEN) compared to rituximab monotherapy (R-mono) for patients with previously treated follicular lymphoma (FL) was investigated in AUGMENT (NCT01938001). Our aim was to evaluate the cost-effectiveness of R-LEN versus R-mono in this setting from a Dutch perspective.Methods: Cost-effectiveness was assessed through a partitioned survival model with three health states from three perspectives (i.e. societal, healthcare and societal including future non-medical costs). Patient-level data from AUGMENT informed effectiveness parameters (i.e. long-term survival) and health state utilities. Resource use and prices were based on AUGMENT and the literature. Clinical experts validated efficacy input parameters and results. Uncertainty was explored through sensitivity and scenario analyses.Results: R-LEN resulted in 1.7 incremental discounted QALYs. Total incremental discounted costs were 67,161 EUR from a societal perspective, resulting in an incremental cost-effectiveness ratio (ICER) of 40,493 EUR/QALY (healthcare: 37,951 EUR/QALY; societal including future non-medical costs: 49,296 EUR/QALY). Sensitivity analyses indicated some uncertainty in the model results. In some scenarios R-LEN exceed an ICER of 50,000 EUR/QALY.Conclusion: R-LEN was cost-effective at a willingness-to-pay (WTP) threshold of 50,000 EUR/QALY in the base-case analyses. Scenario and sensitivity analyses indicated some level of uncertainty regarding this conclusion, depending on the chosen WTP-threshold and perspective.


2018 ◽  
Vol 36 (4_suppl) ◽  
pp. 800-800 ◽  
Author(s):  
Sebastian Stintzing ◽  
Ilse van Oostrum ◽  
Chris Pescott ◽  
Alma Katharina Steinbach-Buechert ◽  
Bart Heeg ◽  
...  

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]


2020 ◽  
Vol 14 (8) ◽  
Author(s):  
Anna Parackal ◽  
Jean-Eric Tarride ◽  
Feng Xie ◽  
Gord Blackhouse ◽  
Jennifer Hoogenes ◽  
...  

Introduction: Recent health technology assessments (HTAs) of robot-assisted radical prostatectomy (RARP) in Ontario and Alberta, Canada, resulted in opposite recommendations, calling into question whether benefits of RARP offset the upfront investment. Therefore, the study objectives were to conduct a cost-utility analysis from a Canadian public payer perspective to determine the cost-effectiveness of RARP. Methods: Using a 10-year time horizon, a five-state Markov model was developed to compare RARP to open radical prostatectomy (ORP). Clinical parameters were derived from Canadian observational studies and a recently published systematic review. Costs, resource utilization, and utility values from recent Canadian sources were used to populate the model. Results were presented in terms of increment costs per quality-adjusted life years (QALYs) gained. A probabilistic analysis was conducted, and uncertainty was represented using cost-effectiveness acceptability curves (CEACs). One-way sensitivity analyses were also conducted. Future costs and QALYs were discounted at 1.5%. Results: Total cost of RARP and ORP were $47 033 and $45 332, respectively. Total estimated QALYs were 7.2047 and 7.1385 for RARP and ORP, respectively. The estimated incremental cost-utility ratio (ICUR) was $25 704 in the base-case analysis. At a willingness-to-pay threshold of $50 000 and $100 000 per QALY gained, the probability of RARP being cost-effective was 0.65 and 0.85, respectively. The model was most sensitive to the time horizon. Conclusions: The results of this analysis suggest that RARP is likely to be cost-effective in this Canadian patient population. The results are consistent with Alberta’s HTA recommendation and other economic evaluations, but challenges Ontario’s reimbursement decision.


2020 ◽  
Vol 12 (1) ◽  
pp. e2020029
Author(s):  
Jialian Li

Background: The four most commonly used chelation regimens for β-thalassemia major patients in China are a combination therapy of deferoxamine and deferiprone (DFO+DFP), deferoxamine(DFO) monotherapy, deferiprone(DFP) monotherapy and deferasirox(DFX) monotherapy. Such patients use iron chelators their whole lives, resulting in enormous treatment costs. This study analyses the cost-utility of these four regimens from the Chinese healthcare system perspective. Methods: A Markov decision model was used over a 70-year time horizon and was populated using clinical data from a systematic literature review. We obtained utility data from local and previous research. Costs were estimated using Chinese national sources. Results: From the base-case analysis results, DFP was the most cost-effective chelation regimen, followed by DFO+DFP, DFO and DFX. DFP had a 99.60%, 78.10% and 64.40% likelihood of being cost-effective versus DFX, DFO and DFO+DFP, respectively, at a payment threshold of 193,932.00 CNY/QALY. Conclusions: DFP was the most cost-effective chelation regimen for β-thalassemia major patients, followed by DFO+DFP, DFO and DFX. Using DFP as the primary treatment regimen may potentially result in cost-savings and QALY gains for the Chinese healthcare system. To increase these benefits, the Chinese government and clinicians should lower drug costs, increase drug utility and reduce mortality and morbidity. Changes in influential parameters easily affect the results of DFO+DFP versus DFP and of DFP versus DFO; clinicians should focus on such parameters and adjust the regimens accordingly.


2018 ◽  
Vol 34 (S1) ◽  
pp. 20-21
Author(s):  
Shaun Harris ◽  
Deborah Fitzsimmons ◽  
Roshan das Nair ◽  
Lucy Bradshaw

Introduction:People with traumatic brain injuries (TBIs) commonly report memory impairments which are persistent, debilitating, and reduce quality of life. As part of the Rehabilitation of Memory in Brain Injury trial, a cost-effectiveness analysis was undertaken to examine the comparative costs and effects of a group memory rehabilitation program for people with TBI.Methods:Individual-level cost and outcome data were collected. Patients were randomized to usual care (n=157) or usual care plus memory rehabilitation (n=171). The primary outcome for the economic analysis was the EuroQol-5D quality of life score at 12 months. A UK NHS costing perspective was used. Missing data was addressed by multiple imputation. One-way sensitivity analyses examined the impact of varying different parameters, and the impact of available cases, on base case findings whilst non-parametric bootstrapping examined joint uncertainty.Results:At 12 months, the intervention was GBP 26.89 (USD 35.76) (SE 249.15) cheaper than usual care; but this difference was statistically non-significant (p=0.914). At 12 months, a QALY loss of −0.007 was observed in the intervention group confidence interval (95% CI: −0.025–0.012) and a QALY gain seen in the usual care group 0.004 (95% CI: -0.017–0.025). This difference was not statistically significant (p=0.442). The base case analysis gave an ICER of GBP 2,445 (USD 3,252) reflecting that the intervention was less effective and less costly compared to usual care. Sensitivity analyses illustrated considerable uncertainty. When joint uncertainty was examined, the probability of the intervention being cost-effective at a willingness-to-pay threshold of GBP 20,000 per QALY gain was 29 percent and 24 percent at GBP 30,000.Conclusions:Our cost-utility analysis indicates that memory rehabilitation was cheaper but less effective than usual care but these findings must be interpreted in the light of small statistically non–significant differences and considerable uncertainty was evident. The ReMemBrIn intervention is unlikely to be considered cost-effective for people with TBI.


2018 ◽  
Vol 34 (S1) ◽  
pp. 120-120
Author(s):  
John Wong ◽  
Stephanie Anne Co ◽  
Joy Bagas ◽  
Ma. Sophia Graciela Reyes ◽  
Hadrian Lim ◽  
...  

Introduction:Colorectal cancer (CRC) is the fourth leading cause of cancer deaths in the Philippines. In 2014, the Philippine Health Insurance Corporation (PhilHealth) created a CRC treatment package. The study aimed to determine the cost-utility and budget impact of CRC screening strategies.Methods:A discrete-event microsimulation model was used to simulate four screening modalities: (i) guaiac-fecal occult blood test (gFOBT) followed by colonoscopy every 10 years; (ii) fecal immunochemical test (FIT) followed by colonoscopy every 10 years; (iii) FIT followed by flexible sigmoidoscopy; and (iv) colonoscopy screening every 10 years. These interventions were all compared to no screening. Parameter values were taken from a rapid review of the medical literature and primary data collection from a nationally representative sample of tertiary hospitals.Results:All screening modalities were very cost effective considering that the incremental cost-effective ratios (ICERs) were lower than the gross domestic product per capita threshold suggested by the World Health Organization. Sensitivity analysis showed that the ICERs of all screening modalities evaluated remained below this threshold. The strategy of using FIT followed by colonoscopy every 10 years had an ICER of USD 6,025, with an annual budget impact of USD 6.5 million, assuming low compliance. With moderate compliance this could increase to USD 18.7 million annually.Conclusions:PhilHealth may introduce a benefit package for outpatient screening of colorectal cancer using the screening modality of annual FIT followed by colonoscopy every 10 years.


2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e18331-e18331
Author(s):  
Jacques Raphael ◽  
Zhuolu Sun ◽  
Georg A. Bjarnason ◽  
Beate Sander ◽  
David M Naimark

e18331 Background: Nivolumab was recently shown to improve overall survival (OS) and health-related quality of life compared to Everolimus in metastatic renal cell carcinoma (mRCC) patients previously treated with antiangiogenic therapies (CheckMate-025 trial). The aim of this study is to assess the cost-utility of Nivolumab versus Everolimus from the perspective of the Canadian publicly funded healthcare system. Methods: To evaluate the cost-utility of Nivolumab versus Everolimus, a Markov cohort model that incorporated data from the phase 3 CheckMate-025 trial and other sources was developed. The outcomes of interest were healthcare costs, life-months and quality-adjusted life-months (QALMs) gained with Nivolumab as well as the incremental cost-effectiveness ratio (ICER), and the incremental net monetary benefit. A lifetime time horizon was used in the base case with costs and outcomes discounted 5% annually. The probabilities of progression and death from cancer and utility values were captured from the CheckMate-025 trial. Expected costs were based on Ontario fees and other sources. Scenario and sensitivity analyses (SAs) were conducted to assess uncertainty. Results: Compared to Everolimus, treatment with Nivolumab provided an additional 3.9 QALMs at an incremental cost of 33,386 Canadian dollars (CAD). The resulting ICER was 8,608CAD per QALM gained. With a willingness-to-pay (WTP) of 50,000CAD per Quality-adjusted life-year (QALY) ( = 4,167CAD per QALM), Nivolumab was not cost-effective in the base case. In one-way SAs, Nivolumab cost, median OS and treatment duration on Nivolumab were sensitive to changes with plausible threshold values. Assuming a WTP of 100,000CAD per QALY ( = 8,334CAD per QALM) and a scenario of Nivolumab cost with no drug wastage, Nivolumab became a cost-effective strategy with an ICER of 7,881CAD per QALM. Conclusions: With its current price , Nivolumab is unlikely to be cost-effective compared with Everolimus for previously treated mRCC patients from a Canadian healthcare payer perspective. While mRCC patients derive a meaningful clinical benefit from Nivolumab, considerations should be given to reduce drug wastage and increase the WTP threshold to render this strategy more affordable.


10.36469/9903 ◽  
2015 ◽  
Vol 2 (2) ◽  
pp. 192-206 ◽  
Author(s):  
Anke van Engen ◽  
Montserrat Casamayor ◽  
Fidelma Loftus ◽  
Martin Coen ◽  
Andy Garnham ◽  
...  

Background: Clostridium difficile is associated with 20–30% of cases of antibiotic-associated diarrhoea. The incidence of C. difficile infection (CDI) is higher in Ireland than in other countries in Europe, and it is associated with considerable morbidity. Previously recommended standard therapeutic options were vancomycin and metronidazole, but the macrocyclic antibiotic fidaxomicin has recently been recommended for use in adults with CDI in Ireland. Objectives: To perform a cost-utility analysis of fidaxomicin compared to oral metronidazole (used to treat initial non-severe disease and first non-severe recurrence) and oral vancomycin (used to treat severe disease and any non-severe recurrence beyond the first) for the treatment of CDI. Methods: A Markov model was used to determine the cost-utility of fidaxomicin in the treatment of all adult CDI patients (base case), patients with severe CDI and patients with initial CDI recurrences, respectively. Patients enter the model in the CDI health state and are treated either with fidaxomicin or current standard of care (oral metronidazole for non-severe CDI; vancomycin for severe CDI) for 10 days. The time horizon was 1 year. Deterministic and probabilistic sensitivity analyses were performed. Health state utilities were derived from the literature. The perspective was that of the Irish Health Service Executive (HSE). Results: In the base case, fidaxomicin was dominant to current standard-of-care therapy, with cost savings of €2,904 and incremental quality-adjusted life year (QALY) gain of 0.031. The main drivers of costeffectiveness were recurrence rates and cost of hospitalization. Fidaxomicin was also dominant for all patient subgroups. The probability of fidaxomicin being cost-effective in all patients with CDI at a willingness to pay threshold of €45,000 per QALY gained was 82%. Conclusion: Fidaxomicin was dominant to the current standard-of-care therapy for CDI. Based on this analysis, fidaxomicin has received reimbursement for CDI treatment under the High Tech Drug Scheme in Ireland.


2021 ◽  
Author(s):  
Antonio Maria Fea ◽  
Francesco Cattel ◽  
Stefano Gandolfi ◽  
Giorgio Buseghin ◽  
Gianluca Furneri ◽  
...  

Abstract BackgroundGlaucoma is a disease characterized by progressive damage of the optic nerve. Several therapeutic options are available to lower intraocular pressure (IOP). In primary open-angle glaucoma (POAG) patients with inadequate IOP control (or controlled with multiple medical therapies or for whom medical therapy is contraindicated), the implantation of micro-invasive glaucoma surgery devices (MIGS) and concomitant cataract surgery has proved to be more effective in reducing intraocular pressure (IOP), as compared to cataract surgery alone. The objective of this study is to assess the cost-effectiveness of iStent inject® device with concurrent cataract surgery vs. cataract surgery alone, in patients with mild-to-moderate POAG, adopting the Italian National Health Service (NHS) perspective.MethodsSimulation of outcomes and costs was undertaken using a Markov model with 4 health states and one-month cycles, that is used to simulate the prognosis of these patients. Efficacy data were obtained from the randomized clinical trial (RCT). A lifetime horizon was adopted in the analysis. A discount rate of 3.5% was applied to both costs and effects. The Italian National Healthcare Service (NHS) perspective was considered, therefore only healthcare direct costs (acquisition of main interventions and subsequent procedures; medications; monitoring and follow-up; adverse events). Model robustness was tested through sensitivity analyses. ResultsResults of the base-case analysis showed that the total lifetime costs were higher in the iStent inject® + concurrent cataract surgery, compared with the cataract surgery alone group (€8,368.51 vs. €7,134.71 respectively). iStent inject® + concurrent cataract surgery was cost-effective vs. cataract surgery alone, with an incremental cost-effectiveness ratio of €13,037.01 per quality-adjusted life year (QALY) gained. Both one-way deterministic and probabilistic sensitivity analyses confirmed robustness of base-case results. The acceptability curve of cost-effectiveness (CEAC) analysis showed that iStent inject® + cataract surgery would have a 98% probability of being cost-effective, compared to cataract surgery alone, when the willingness to pay (WTP) is equal to €50,000 per QALY gained.ConclusionsThe results of the cost-utility analysis confirm that iStent inject® + cataract surgery is a cost-effective option for the treatment of patients affected by mild-to-moderate POAG, compared with cataract surgery alone, when evaluated from the Italian NHS perspective. Trial registration: Not applicable


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