Modeling the cost-effectiveness of a new treatment for MS (natalizumab) compared with current standard practice in Sweden

2008 ◽  
Vol 14 (5) ◽  
pp. 679-690 ◽  
Author(s):  
G Kobelt ◽  
J Berg ◽  
P Lindgren ◽  
B Jonsson ◽  
L Stawiarz ◽  
...  

Objective To estimate the cost-effectiveness of a new treatment (natalizumab) for multiple sclerosis (MS) compared with current standard therapy with disease-modifying drugs (DMDs) in Sweden. Methods A Markov model was constructed to illustrate disease progression based on functional disability (the Expanded Disability Status Scale (EDSS)). The effectiveness of natalizumab was based on a 2-year clinical trial in 942 patients (AFFIRM). The effectiveness of current DMDs was estimated from a matched sample of 512 patients in the Stockholm MS registry. Patients withdrawing from treatment were assumed to follow the disease course of 824 patients with relapsing–remitting disease at onset in the Ontario natural history cohort. Costs and utilities are based on a recent observational study in 1339 patients. All data sets were available at the patient level. Main results are presented from the societal perspective, over a 20-year time frame, in 2005 Euros (€1 = 9.25 SEK). Results In the base case, treatment with natalizumab was less expensive and more effective than treatment with current DMDs. When only healthcare costs were considered, the cost per quality-adjusted life year gained with natalizumab was €38 145. Results are sensitive only to the time horizon of the analysis and assumptions about effectiveness of natalizumab beyond the trial. Conclusions This cost-effectiveness analysis used registry data, cohort and observational studies to extrapolate the efficacy findings of natalizumab from the AFFIRM clinical trial to measure effectiveness in clinical practice. The analysis results suggest that for the population considered, natalizumab provides an additional health benefit at a similar cost to current DMDs from a societal perspective.

2011 ◽  
Vol 27 (3) ◽  
pp. 193-200 ◽  
Author(s):  
Gisela Kobelt ◽  
Ingrid Lekander ◽  
Andrea Lang ◽  
Bernd Raffeiner ◽  
Costantino Botsios ◽  
...  

Objectives: To explore the cost-effectiveness of early biologic treatment, followed by dose-reduction in the case of remission, of active rheumatoid arthritis (RA), compared with standard treatment with methotrexate (MTX) in Sweden.Methods: Effectiveness (function, disease activity, erosions) in early RA for both alternatives was taken from a clinical trial comparing etanercept (ETA) combined with MTX to MTX alone. Patients discontinuing treatment can switch to another or their first biologic treatment. For patients in remission (Disease Activity Score [DAS28] < 2.6), ETA is reduced to half the dose. Return to full dose occurs when DAS28 reaches ≥ 3.2 again. Costs and utilities by level of functional capacity from an observational study are used. The model is analyzed as a micro-simulation and results are presented from the societal perspective for Sweden, for 10 years; costs (€2008) and effects are discounted at 3 percent. Sensitivity analysis was performed for the perspective, the time horizon, switching, and dose-reduction.Results: The main analysis conservatively assumes 50 percent switching at discontinuation. The cost per quality-adjusted life-year (QALY) gained with early ETA/MTX treatment is €13,500 (societal perspective, incremental cost of €15,500 and incremental QALYs of 1.15). With 75 percent switching, the cost per QALY gained was €10,400. Over 20 years, the cost per QALY gained was €8,200. Results were further sensitive to the time patients remained on half dose and the perspective.Conclusions and Policy Implications: This study combines clinical trial and clinical practice data to explore cost-effective treatment scenarios in early RA, including the use of biologics. Our results indicate that a situation where a considerable proportion of patients achieve remission, dose-adjustments will increase the cost-effectiveness of treatment.


Author(s):  
Lorenzo Giovanni Mantovani ◽  
Gianluca Furneri ◽  
Rossella Bitonti ◽  
Paolo Cortesi ◽  
Elisa Puma ◽  
...  

BACKGROUND: Delayed-release dimethyl fumarate (also known as gastro-resistant dimethyl fumarate, hereafter dimethyl fumarate) is an oral disease-modifying therapy used for the treatment of Relapsing-Remitting Multiple Sclerosis (RRMS), an autoimmune chronic inflammatory condition of the central nervous system.OBJECTIVE: The objective of this economic analysis was to compare cost-effectiveness of dimethyl fumarate with the alternatives used as first-line treatment of RRMS in Italy.METHODS: The analysis was conducted from the Italian societal perspective. Health outcomes and costs were evaluated over a 50-year time horizon (equivalent to a lifetime horizon). Both health outcomes and costs were discounted at 3.5%. The cost-effectiveness analysis was conducted by adapting a Markov model, already used in previous similar economic analyses conducted in RRMS, to the Italian context. The Markov model estimated the clinical and economic consequences of treating RRMS patients with the following therapeutic options: dimethyl fumarate; interferon (IFN) beta-1a subcutaneous (SC) at two different doses, 22 mcg and 44 mcg; IFN beta-1b SC; glatiramer acetate (GA) SC 20 mg; oral teriflunomide. Clinical efficacy data were retrieved from an elaboration of an already published mixed treatment comparison (MTC). Both direct and indirect costs (disability, treatment acquisition, administration, monitoring, relapses, adverse events) were included in the analysis. One-way and probabilistic sensitivity analyses were carried out and cost-effectiveness acceptability curves generated.RESULTS: In the base-case analysis, dimethyl fumarate was more efficacious than alternatives, in terms of both survival (19.634 vs. 19.440-19.600 life years for alternatives), and quality-of-life-adjusted survival (6.526 vs. 5.143- 6.189 QALYs for alternatives). The total lifetime cost per patient treated with dimethyl fumarate (€ 954,286) was lower than that of the other DMTs included in the analysis. Therefore, dimethyl fumarate was dominant compared with all analyzed alternatives. Dimethyl fumarate was also the therapeutic option with the highest benefit on disease burden. In fact, costs of disability management were lower than those of all the other first-line drugs included in the analysis. The results of one-way deterministic sensitivity analysis and probabilistic sensitivity analysis confirmed the reliability of base-case results.CONCLUSIONS: The results of the cost-effectiveness analysis confirm that dimethyl fumarate is an optimal first-line treatment for RRMS in Italy, compared with the other first-line alternatives included in the economic analysis, when evaluated from the societal perspective.


2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Weiyi Ni ◽  
Jia Liu ◽  
Yawen Jiang ◽  
Jing Wu

Abstract Background Clinical trials in China have demonstrated that ranibizumab can improve the clinical outcomes of branch retinal vein occlusion (BRVO) and central retinal vein occlusion (CRVO). However, no economic evaluation of ranibizumab has been conducted among Chinese patient population. Methods To provide insights into the economic profile of ranibizumab among Chinese RVO population, a Markov state-transition model was used to predict the outcomes of ranibizumab comparing to laser photocoagulation and observational-only care from the societal perspective. This model simulated changes in patient visuality, quality-adjusted of life years (QALY), medical costs, and direct non-medical costs of individuals with visual impairment due to BRVO or CRVO in lifetime. The base-case analysis used an annual discount rate of 5% for costs and benefits following the China Guidelines for Pharmacoeconomic Evaluations. Deterministic and probabilistic sensitivity analyses were performed to test the robustness of the model. Results The base-case incremental cost-effectiveness ratio (ICER) comparing ranibizumab to laser photocoagulation was ¥65,008/QALY among BRVO patients and was ¥65,815/QALY among CRVO patients, respectively. Comparing to the 2019 gross domestic product (GDP) per capita of ¥71,000, both two ICERs were far below the cost-effective threshold at three times of GDP per capita (¥213,000). The deterministic and probabilistic sensitivity analyses demonstrated the base-case results were robust in most of the simulation scenarios. Conclusion The current Markov model demonstrated that ranibizumab may be cost-effective compared with laser photocoagulation to treat BRVO and cost-effective compared to observation-only care to treat CRVO in China from the societal perspective.


2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Mireia Massot Mesquida ◽  
Frans Folkvord ◽  
Gemma Seda ◽  
Francisco Lupiáñez-Villanueva ◽  
Pere Torán Monserrat

Abstract Background Growing evidence shows the effects of psychotropic drugs on the evolution of dementia. Until now, only a few studies have evaluated the cost-effectiveness of psychotropic drugs in institutionalized dementia patients. This study aims to assess the cost-utility of intervention performed in the metropolitan area of Barcelona (Spain) (MN) based on consensus between specialized caregivers involved in the management of dementia patients for optimizing and potentially reducing the prescription of inappropriate psychotropic drugs in this population. This analysis was conducted using the Monitoring and Assessment Framework for the European Innovation Partnership on Active and Healthy Ageing (MAFEIP) tool. Methods The MAFEIP tool builds up from a variety of surrogate endpoints commonly used across different studies in order to estimate health and economic outcomes in terms of incremental changes in quality adjusted life years (QALYs), as well as health and social care utilization. Cost estimates are based on scientific literature and expert opinion; they are direct costs and include medical visits, hospital care, medical tests and exams and drugs administered, among other concepts. The healthcare costs of patients using the intervention were calculated by means of a medication review that compared patients’ drug-related costs before, during and after the use of the intervention conducted in MN between 2012 and 2014. The cost-utility analysis was performed from the perspective of a health care system with a time horizon of 12 months. Results The tool calculated the incremental cost-effectiveness ratio (ICER) of the intervention, revealing it to be dominant, or rather, better (more effective) and cheaper than the current (standard) care. The ICER of the intervention was in the lower right quadrant, making it an intervention that is always accepted even with the lowest given Willingness to Pay (WTP) threshold value (€15,000). Conclusions The results of this study show that the intervention was dominant, or rather, better (more effective) and cheaper than the current (standard) care. This dominant intervention is therefore recommended to interested investors for systematic application.


2018 ◽  
Vol 36 (07) ◽  
pp. 678-687 ◽  
Author(s):  
Catherine M. Albright ◽  
Erika F. Werner ◽  
Brenna L. Hughes

Objective To determine threshold cytomegalovirus (CMV) infectious rates and treatment effectiveness to make universal prenatal CMV screening cost-effective. Study Design Decision analysis comparing cost-effectiveness of two strategies for the prevention and treatment of congenital CMV: universal prenatal serum screening and routine, risk-based screening. The base case assumptions were a probability of primary CMV of 1% in seronegative women, hyperimmune globulin (HIG) effectiveness of 0%, and behavioral intervention effectiveness of 85%. Screen-positive women received monthly HIG and screen-negative women received behavioral counseling to decrease CMV seroconversion. The primary outcome was the cost per maternal quality-adjusted life year (QALY) gained with a willingness to pay of $100,000 per QALY. Results In the base case, universal screening is cost-effective, costing $84,773 per maternal QALY gained. In sensitivity analyses, universal screening is cost-effective only at a primary CMV incidence of more than 0.89% and behavioral intervention effectiveness of more than 75%. If HIG is 30% effective, primary CMV incidence can be 0.82% for universal screening to be cost-effective. Conclusion The cost-effectiveness of universal maternal screening for CMV is highly dependent on the incidence of primary CMV in pregnancy. If efficacious, HIG and behavioral counseling allow universal screening to be cost-effective at lower primary CMV rates.


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.


2020 ◽  
Vol 24 (2) ◽  
pp. 1-180 ◽  
Author(s):  
Nigel Fleeman ◽  
Rachel Houten ◽  
Adrian Bagust ◽  
Marty Richardson ◽  
Sophie Beale ◽  
...  

Background Thyroid cancer is a rare cancer, accounting for only 1% of all malignancies in England and Wales. Differentiated thyroid cancer (DTC) accounts for ≈94% of all thyroid cancers. Patients with DTC often require treatment with radioactive iodine. Treatment for DTC that is refractory to radioactive iodine [radioactive iodine-refractory DTC (RR-DTC)] is often limited to best supportive care (BSC). Objectives We aimed to assess the clinical effectiveness and cost-effectiveness of lenvatinib (Lenvima®; Eisai Ltd, Hertfordshire, UK) and sorafenib (Nexar®; Bayer HealthCare, Leverkusen, Germany) for the treatment of patients with RR-DTC. Data sources EMBASE, MEDLINE, PubMed, The Cochrane Library and EconLit were searched (date range 1999 to 10 January 2017; searched on 10 January 2017). The bibliographies of retrieved citations were also examined. Review methods We searched for randomised controlled trials (RCTs), systematic reviews, prospective observational studies and economic evaluations of lenvatinib or sorafenib. In the absence of relevant economic evaluations, we constructed a de novo economic model to compare the cost-effectiveness of lenvatinib and sorafenib with that of BSC. Results Two RCTs were identified: SELECT (Study of [E7080] LEnvatinib in 131I-refractory differentiated Cancer of the Thyroid) and DECISION (StuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine-refractory thyrOid caNcer). Lenvatinib and sorafenib were both reported to improve median progression-free survival (PFS) compared with placebo: 18.3 months (lenvatinib) vs. 3.6 months (placebo) and 10.8 months (sorafenib) vs. 5.8 months (placebo). Patient crossover was high (≥ 75%) in both trials, confounding estimates of overall survival (OS). Using OS data adjusted for crossover, trial authors reported a statistically significant improvement in OS for patients treated with lenvatinib compared with those given placebo (SELECT) but not for patients treated with sorafenib compared with those given placebo (DECISION). Both lenvatinib and sorafenib increased the incidence of adverse events (AEs), and dose reductions were required (for > 60% of patients). The results from nine prospective observational studies and 13 systematic reviews of lenvatinib or sorafenib were broadly comparable to those from the RCTs. Health-related quality-of-life (HRQoL) data were collected only in DECISION. We considered the feasibility of comparing lenvatinib with sorafenib via an indirect comparison but concluded that this would not be appropriate because of differences in trial and participant characteristics, risk profiles of the participants in the placebo arms and because the proportional hazard assumption was violated for five of the six survival outcomes available from the trials. In the base-case economic analysis, using list prices only, the cost-effectiveness comparison of lenvatinib versus BSC yields an incremental cost-effectiveness ratio (ICER) per quality-adjusted life-year (QALY) gained of £65,872, and the comparison of sorafenib versus BSC yields an ICER of £85,644 per QALY gained. The deterministic sensitivity analyses show that none of the variations lowered the base-case ICERs to < £50,000 per QALY gained. Limitations We consider that it is not possible to compare the clinical effectiveness or cost-effectiveness of lenvatinib and sorafenib. Conclusions Compared with placebo/BSC, treatment with lenvatinib or sorafenib results in an improvement in PFS, objective tumour response rate and possibly OS, but dose modifications were required to treat AEs. Both treatments exhibit estimated ICERs of > £50,000 per QALY gained. Further research should include examination of the effects of lenvatinib, sorafenib and BSC (including HRQoL) for both symptomatic and asymptomatic patients, and the positioning of treatments in the treatment pathway. Study registration This study is registered as PROSPERO CRD42017055516. Funding The National Institute for Health Research Health Technology Assessment programme.


2021 ◽  
Author(s):  
Y. Natalia Alfonso ◽  
Adnan A Hyder ◽  
Olakunle Alonge ◽  
Shumona Sharmin Salam ◽  
Kamran Baset ◽  
...  

Abstract Drowning is the leading cause of death among children 12-59 months old in rural Bangladesh. This study evaluated the cost-effectiveness of a large-scale crèche intervention in preventing child drowning. Estimates of the effectiveness of the crèches was based on prior studies and the program cost was assessed using monthly program expenditures captured prospectively throughout the study period from two different implementing agencies. The study evaluated the cost-effectiveness from both a program and societal perspective. Results showed that from the program perspective the annual operating cost of a crèche was $416.35 (95%C.I.: $222 to $576), the annual cost per child was $16 (95%C.I.: $9 to $22) and the incremental-cost-effectiveness ratio (ICER) per life saved with the crèches was $17,803 (95%C.I.: $9,051 to $27,625). From the societal perspective (including parents time valued) the ICER per life saved was -$176,62 (95%C.I.: -$347,091 to -$67,684)—meaning crèches generated net economic benefits per child enrolled. Based on the ICER per disability-adjusted-life years averted from the societal perspective (excluding parents time), $2,020, the crèche intervention was cost-effective even when the societal economic benefits were ignored. Based on the evidence, the creche intervention has great potential for reducing child drowning at a cost that is reasonable.


Author(s):  
Brendan L Limone ◽  
William L Baker ◽  
Craig I Coleman

Background: A number of new anticoagulants for stroke prevention in atrial fibrillation (SPAF) have gained regulatory approval or are in late-stage development. We sought to conduct a systematic review of economic models of dabigatran, rivaroxaban and apixaban for SPAF. Methods: We searched the Medline, Embase, National Health Service Economic Evaluation Database and Health Technology Assessment database along with the Tuft’s Registry through October 10, 2012. Included models assessed the cost-effectiveness of dabigatran (150mg, 110mg, sequential), rivaroxaban or apixaban for SPAF using a Markov model or discrete event simulation and were published in English. Results: Eighteen models were identified. All models utilized a lone randomized trial (or an indirect comparison utilizing a single study for any given direct comparison), and these trials were clinically and methodologically heterogeneous. Dabigatran 150mg was assessed in 9 of models, dabigatran 110mg in 8, sequential dabigatran in 9, rivaroxaban in 4 and apixaban in 4. Adjusted-dose warfarin (either trial-like, real-world prescribing or genotype-dosed) was a potential first-line therapy in 94% of models. Models were conducted from the perspective of the United States (44%), European countries (39%) and Canada (17%). In base-case analyses, patients typically were at moderate-risk of ischemic stroke, initiated anticoagulation between 65 and 73 years of age, and were followed for or near a lifetime. All models reported cost/quality-adjusted life-year (QALY) gained, and while 22% of models reported using a societal perspective, no model included costs of lost productivity. Four models reported an incremental cost-effectiveness ratio (ICER) for a newer anticoagulant (dabigatran 110mg (n=4)/150mg (n=2); rivaroxaban (n=1)) vs. warfarin above commonly reported willingness-to-pay thresholds. ICERs (in 2012US$) vs. warfarin ranged from $3,547-$86,000 for dabigatran 150mg, $20,713-$150,000 for dabigatran 110mg, $4,084-$21,466 for sequentially-dosed dabigatran and $23,065-$57,470 for rivaroxaban. In addition, apixaban was demonstrated to be an economically dominant strategy compared to aspirin and to be dominant or cost-effective ($11,400-$25,059) vs. warfarin. Based on separate indirect treatment comparison meta-analyses, 3 models compared the cost-effectiveness of these new agents and reported conflicting results. Conclusions: Cost-effectiveness models of newer anticoagulants for SPAF have been extensively published. Models have frequently found newer anticoagulants to be cost-effective, but due to the lack of head-to-head trial comparisons and heterogeneity in clinical characteristic of underlying trials and modeling methods, it is currently unclear which of these newer agents is most cost-effective.


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