scholarly journals Cost-Effectiveness of Multiple Sclerosis Disease-Modifying Therapies: A Systematic Review of the Literature

2012 ◽  
Vol 2012 ◽  
pp. 1-13 ◽  
Author(s):  
David Yamamoto ◽  
Jonathan D. Campbell

Objective. To provide a current and comprehensive understanding of the cost-effectiveness of DMTs for the treatment of MS by quantitatively evaluating the quality of recent cost-effectiveness studies and exploring how the field has progressed from past recommendations.Methods. We assessed the quality of studies that met our systematic literature search criteria using the Quality of Health Economic Studies validated instrument.Results. Of the 82 studies that met our initial search criteria, we included 22 in this review. Four studies (18%) achieved quality category 2, three studies (14%) achieved quality category 3, and 15 studies (68%) achieved the highest quality category 4. 91% of studies were simulation models. 13 studies (59%) had quality-adjusted life years (QALYs) as the primary outcome measure, included a societal perspective in the analysis, and utilized time horizons of 10 years to lifetime.Conclusions. To continue to improve the cost-effectiveness evidence of DMTs, we recommend: lifetime horizons, societal perspectives, and QALYs; supplemental evidence with shorter horizons, payer perspectives, and clinical outcomes to inform multiple decision makers; development of modeling and input standards for comparability; head-to-head RCTs between DMTs and long-term prospective studies; and comprehensive cost-effectiveness studies that compare all appropriate DMTs.

2015 ◽  
Vol 19 (81) ◽  
pp. 1-246 ◽  
Author(s):  
Janine Dretzke ◽  
Deirdre Blissett ◽  
Chirag Dave ◽  
Rahul Mukherjee ◽  
Malcolm Price ◽  
...  

BackgroundChronic obstructive pulmonary disease (COPD) is a chronic progressive lung disease characterised by non-reversible airflow obstruction. Exacerbations are a key cause of morbidity and mortality and place a considerable burden on health-care systems. While there is evidence that patients benefit from non-invasive ventilation (NIV) in hospital during an acute exacerbation, evidence supporting home use for more stable COPD patients is limited. In the UK, domiciliary NIV is considered on health economic grounds in patients after three hospital admissions for acute hypercapnic respiratory failure.ObjectiveTo assess the clinical effectiveness and cost-effectiveness of domiciliary NIV by systematic review and economic evaluation.Data sourcesBibliographic databases, conference proceedings and ongoing trial registries up to September 2014.MethodsStandard systematic review methods were used for identifying relevant clinical effectiveness and cost-effectiveness studies assessing NIV compared with usual care or comparing different types of NIV. Risk of bias was assessed using Cochrane guidelines and relevant economic checklists. Results for primary effectiveness outcomes (mortality, hospitalisations, exacerbations and quality of life) were presented, where possible, in forest plots. A speculative Markov decision model was developed to compare the cost-effectiveness of domiciliary NIV with usual care from a UK perspective for post-hospital and more stable populations separately.ResultsThirty-one controlled effectiveness studies were identified, which report a variety of outcomes. For stable patients, a modest volume of evidence found no benefit from domiciliary NIV for survival and some non-significant beneficial trends for hospitalisations and quality of life. For post-hospital patients, no benefit from NIV could be shown in terms of survival (from randomised controlled trials) and findings for hospital admissions were inconsistent and based on limited evidence. No conclusions could be drawn regarding potential benefit from different types of NIV. No cost-effectiveness studies of domiciliary NIV were identified. Economic modelling suggested that NIV may be cost-effective in a stable population at a threshold of £30,000 per quality-adjusted life-year (QALY) gained (incremental cost-effectiveness ratio £28,162), but this is associated with uncertainty. In the case of the post-hospital population, results for three separate base cases ranged from usual care dominating to NIV being cost-effective, with an incremental cost-effectiveness ratio of less than £10,000 per QALY gained. All estimates were sensitive to effectiveness estimates, length of benefit from NIV (currently unknown) and some costs. Modelling suggested that reductions in the rate of hospital admissions per patient per year of 24% and 15% in the stable and post-hospital populations, respectively, are required for NIV to be cost-effective.LimitationsEvidence on key clinical outcomes remains limited, particularly quality-of-life and long-term (> 2 years) effects. Economic modelling should be viewed as speculative because of uncertainty around effect estimates, baseline risks, length of benefit of NIV and limited quality-of-life/utility data.ConclusionsThe cost-effectiveness of domiciliary NIV remains uncertain and the findings in this report are sensitive to emergent data. Further evidence is required to identify patients most likely to benefit from domiciliary NIV and to establish optimum time points for starting NIV and equipment settings.Future work recommendationsThe results from this report will need to be re-examined in the light of any new trial results, particularly in terms of reducing the uncertainty in the economic model. Any new randomised controlled trials should consider including a sham non-invasive ventilation arm and/or a higher- and lower-pressure arm. Individual participant data analyses may help to determine whether or not there are any patient characteristics or equipment settings that are predictive of a benefit of NIV and to establish optimum time points for starting (and potentially discounting) NIV.Study registrationThis study is registered as PROSPERO CRD42012003286.FundingThe National Institute for Health Research Health Technology Assessment programme.


Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 3336-3336 ◽  
Author(s):  
Amy K. O’Sullivan ◽  
Milton C. Weinstein ◽  
Ankur Pandya ◽  
David Thompson ◽  
Amelia Langston ◽  
...  

Abstract Trial data suggest that posaconazole is similar to fluconazole in preventing invasive fungal infections (IFIs) among allogeneic progenitor cell transplant recipients with graft-versus-host disease (GVHD). We estimated the cost-effectiveness of posaconazole versus fluconazole in this population in the US. A decision-analytic model was developed to estimate the average per patient treatment costs, IFIs avoided, life-years gained, and incremental cost per life-year gained of prophylaxis (2006 US$). The model extrapolates the trial results to a lifetime horizon to include long-term mortality due to GVHD. In the model, patients are assumed to receive posaconazole or fluconazole; efficacy data were obtained from the clinical trial. Long-term mortality and prophylaxis drug and IFI treatment costs were estimated from secondary sources. One-way and probabilistic sensitivity analyses were conducted. Posaconazole is associated with fewer IFIs (0.05 vs. 0.09), increased life years (7.87 vs. 7.66), and higher IFI-related costs (prophylaxis and IFI treatment) ($8,750 vs. $5,530) per patient relative to fluconazole. Costs for treatment of IFIs comprised 95% of the total cost for fluconazole and 35% for posaconazole. The incremental cost-effectiveness of posaconazole versus fluconazole is estimated to be $15,700 per life-year saved. Results are most sensitive to changes in the cost of treating an IFI and the efficacy of prophylaxis. Results from the probabilistic analysis indicate that there is an 88% probability that posaconazole is cost-effective at a $50,000 per life year saved threshold. We conclude that posaconazole is a cost-effective strategy for the prevention of IFIs in patients with GVHD.


2009 ◽  
Vol 35 (5) ◽  
pp. 761-769 ◽  
Author(s):  
Carol A. Brownson ◽  
Thomas J. Hoerger ◽  
Edwin B. Fisher ◽  
Kerry E. Kilpatrick

Purpose The purpose of this study is to estimate the cost-effectiveness of diabetes self-management programs in real-world community primary care settings. Estimates incorporated lifetime reductions in disease progression, costs of adverse events, and increases in quality of life. Methods Clinical results and costs were based on programs of the Diabetes Initiative of the Robert Wood Johnson Foundation, implemented in primary care and community settings in disadvantaged areas with notable health disparities. Program results were used as inputs to a Markov simulation model to estimate the long-term effects of self-management interventions. A health systems perspective was adopted. Results The simulation model estimates that the intervention does reduce discounted lifetime treatment and complication costs by $3385, but this is more than offset by the $15 031 cost of implementing the intervention and maintaining its effects in subsequent years. The intervention is estimated to reduce long-term complications, leading to an increase in remaining life-years and quality-adjusted life-years (QALYs). The incremental cost-effectiveness ratio is $39 563/QALY, well below a common benchmark of $50 000/QALY. Sensitivity analyses tested the robustness of the model’s estimates under various alternative assumptions. The model generally predicts acceptable cost-effectiveness ratios. Conclusions Self-management programs for type 2 diabetes are cost-effective from a health systems perspective when the cost savings due to reductions in long-term complications are recognized. These findings may justify increased reimbursement for effective self-management programs in diverse settings.


2006 ◽  
Vol 22 (4) ◽  
pp. 469-477 ◽  
Author(s):  
Patrik Sobocki ◽  
Mattias Ekman ◽  
Hans Ågren ◽  
Bengt Jönsson ◽  
Clas Rehnberg

Objectives: The objective of this study was to develop a model to assess the cost-effectiveness of a new treatment for patients with depression.Methods: A Markov simulation model was constructed to evaluate standard care for depression as performed in clinical practice compared with a new treatment for depression. Costs and effects were estimated for time horizons of 6 months to 5 years. A naturalistic longitudinal observational study provided data on costs, quality of life, and transition probabilities. Data on long-term consequences of depression and mortality risks were collected from the literature. Cost-effectiveness was quantified as quality-adjusted life-years (QALYs) gained from the new treatment compared with standard care, and the societal perspective was taken. Probabilistic analyses were conducted to present the uncertainty in the results, and sensitivity analyses were conducted on key parameters used in the model.Results: Compared with standard care, the new hypothetical therapy was predicted to substantially decrease costs and was also associated with gains in QALYs. With an improved treatment effect of 50 percent on achieving full remission, the net cost savings were 20,000 Swedish kronor over a 5-year follow-up time, given equal costs of treatments. Patients gained .073 QALYs over 5 years. The results are sensitive to changes in assigned treatment effects.Conclusions: The present study provides a new model for assessing the cost-effectiveness of treatments for depression by incorporating full remission as the treatment goal and QALYs as the primary outcome measure. Moreover, we show the usefulness of naturalistic real-life data on costs and quality of life and transition probabilities when modeling the disease over time.


Author(s):  
Giovanni Di Minno ◽  
Roberto Ravasio

Objectives: To evaluate the cost-effectiveness analysis (CEA) of caplacizumab in combination with plasmapheresis (PEX) and immunosuppression compared to PEX and immunosuppression in the treatment of acute episodes of iTTP. Methods: A Markov model was used to conduct the CEA from the perspective of the hospital, over a lifetime horizon. Clinical data derived from HERCULES trial and a systematic literature review. Economic input included direct costs only. Utility and disutility values were obtained from literature. Data on healthcare resources and costs were retrieved from HERCULES trial, literature, TTP guidelines and Italian tariffs. A sensitivity analysis was conducted. The cost-effectiveness probability was tested for several options of discount levels considering a suggested willingness to pay (WTP) threshold of € 60,000 in Italy. Results: The use of caplacizumab in combination with PEX and immunosuppression is associated with a positive difference in survival of 3.27 life years (24.53 vs 21.26) and in quality of life of 3.06 QALY (22.01 vs 18.96) when compared to PEX and immunosuppression. Caplacizumab leads to an ICER per life years of € 41,653 and an ICER per QALY of € 44,572. For the suggested WTP threshold, the probability of caplacizumab being cost-effective is 82.4% (no discount), 92.8% (15% discount), 95.3% (20% discount), 96.9% (25% discount) and 98.2% (30% discount). Conclusions: Caplacizumab in addition to PEX and immunosuppression is cost-effective, allowing the hospital to achieve greater efficiency in managing the burden of a life-threatening disease such as iTTP.


2009 ◽  
Vol 2 (1) ◽  
pp. 1-9 ◽  
Author(s):  
Jenna L. Yoder ◽  
Khalid M. Kamal

Objectives: To explore the use of pharmacoeconomic principles through examination of economic evaluations pertaining to the combination of the monoclonal antibody rituximab with conventional CHOP (cyclophosphamide/doxorubicin/ vincristine/ prednisone) or CHOP-like chemotherapy regimens in patients with non-Hodgkin lymphoma. Methods: A literature search was conducted using Evidence-Based Medical Reviews (EBMR), International Pharmaceutical Abstracts (IPA), and Medline databases to identify all economic studies relating to rituximab in combination with CHOP or CHOP-like regimens. The systematic evaluation also utilized the Quality of Health Economic Studies instrument to assess the quality of each study that was included in the final review. Results: Initially, eight studies were retrieved which included the use of rituximab in non-Hodgkin lymphoma treatment. Of these, four studies were excluded as rituximab was used as a stand-alone treatment option. The remaining four studies involved conventional CHOP therapy versus the combination with rituximab (R-CHOP) in patients with non-Hodgkin lymphoma. One study employed a cost-effectiveness analysis while the remaining three studies used a cost-utility analysis and reported the outcomes in terms of quality-adjusted life years (QALYs). Conclusions: The cost-effectiveness evaluation illustrated the dominance of R-CHOP over CHOP-alone in terms of both lower costs and increased life years gained. The cost-utility of R-CHOP in terms of costs/QALYs were below the accepted threshold of 50,000 in international monetary units. Through examination of evaluation principles employed, it is found that valid results are highly dependent on the input data, assumptions, and sensitivity analyses. Clinical decisionmakers must take into account specific inclusions of costs relevant to their own practice setting.


2016 ◽  
Vol 34 (7_suppl) ◽  
pp. 23-23
Author(s):  
Sarina Isenberg ◽  
David R Holtgrave ◽  
Chunhua Lu ◽  
John P McQuade ◽  
Brian Weir ◽  
...  

23 Background: The objectives of the study were to determine whether a Palliative Care Unit (PCU) provides benefits not just from a cost perspective, but from a patient and caregiver quality of life (QOL) perspective. Methods: (1) Calculate the total costs of the PCU; (2) Leverage a threshold analysis to estimate the Quality-Adjusted Life Years (QALYs) required for the PCU to be cost effective; and (3) Determine whether it is feasible for the program to yield the required number of QALYs. Setting was the Johns Hopkins Health System Palliative Care Unit (PCU) in Baltimore, MD. Analysis was based on patient volume from March 2013-2014. Results: There were 209 palliative patients. The costs for the societal perspective was $2,044,364 and the required number of QALYs to deem it cost effective were 11.36. The net costs for the hospital perspective was $625,777 (gross cost was $993,528; however, the program generated $367,751 in savings for the hospital through treating patients in the PCU as opposed to other functional units), and the required number of QALYs to deem it cost effective were 3.48. To determine whether the program is able to achieve the number of QALYs required, the study team generated aggregated QALYs based on other studies’ evidence for palliative care’s improvement of quality of life for patients and their caregivers. Combining the QALYs generated from the aggregated calculations for patients (0.12) and caregivers (4.60), the program had the potential to yield a total of 4.73 QALYs. Conclusions: This analysis suggests that the PCU is cost effective from the hospital perspective in the sense that the benefits it provides to patients’ and caregivers’ quality of life outweighs the cost of care. Future studies should continue to evaluate palliative care from a cost effectiveness perspective that incorporates a consideration of the quality of life improvements, rather than just cost-reduction.


2009 ◽  
Vol 15 (6) ◽  
pp. 741-751 ◽  
Author(s):  
G Kobelt ◽  
B Texier-Richard ◽  
P Lindgren

Objective To evaluate the long-term costs and quality of life (QoL) with and without disease-modifying treatments (DMTs) of patients with multiple sclerosis (MS). Methods Data on resource consumption, productivity losses, QoL (utility), and fatigue were collected from 1355 patients registered with a patient association and descriptive analyses was performed. A Markov model was developed to estimate costs and utility over 20 years using the survey data. Disease progression without DMTs was taken from an epidemiological cohort in France (EDMUS cohort, LYON). Progression under DMTs was estimated from the Stockholm MS registry. Results are presented as cost per quality-adjusted life-years (QALYs), from the societal perspective, in EUR2007, discounted at 3%. Results Mean Expanded Disability Status Scale (EDSS) was 4.4 and mean total annual costs per patient were EUR44,400, of which 47% were productivity losses and 11% informal care. Public payers cover an estimated 48% of costs. Mean utility was 0.52, and the loss compared with the normal population was estimated at 0.28. Costs and utility ranged from EUR16,000 and 0.79 at EDSS 1 to EUR76,000 and 0.11 at EDSS 8–9. Over 20 years, costs were estimated at EUR429,000 and QALYs at 8.96 for patients without DMTs and at EUR433,207 and 9.24 QALYs if all patients were starting treated with DMTs at EDSS 1–3. Conclusion Although the data for this analysis come from different sources, the results indicate that the cost increase with DMTs is moderate.


2009 ◽  
Vol 15 (3) ◽  
pp. 679-725
Author(s):  
J. Buckle

ABSTRACTDepression is a significant burden for the United Kingdom economy and despite conclusive evidence on the clinical efficacy of treatments and acknowledgements of the impact on quality of life, a high proportion still goes undiagnosed and untreated. The purpose of this paper is to present the economic case for a more structured approach to depression management, using techniques from the disciplines of health economics and actuarial science to demonstrate cost-effectiveness and return on investment. The results are presented first as an economic cost-effectiveness analysis, comparing the benefits of additional quality-adjusted life years (QALYs) with the costs, and secondly as a financial projection model of costs and savings, familiar to actuaries.The results of the model show that from a societal perspective, disease management programmes for depression are likely to both reduce costs and increase quality of life for patients in the overall adult population. This is also true from the perspective of an employer who has the cost burden of direct medical costs and sickness absence. For a healthcare payer who is not bearing the cost of sickness absence, such as a primary care trust (PCT) or private insurer, disease management programmes are likely to improve quality of life, but increase direct healthcare costs. However, the additional cost per QALY is well below the commonly used threshold in the U.K. of £30,000; therefore, most health economists would deem disease management programmes for severe and moderate depression to be a good use of public healthcare funds. The actuarial calculations, which show an internal rate of return for 45% to 50%, validate this conclusion.


2021 ◽  
Vol 50 (Supplement_1) ◽  
Author(s):  
Yuqi Zhang ◽  
Hannah Carter ◽  
Peter Lazzarini ◽  
Susanna Cramb ◽  
Rosana Pacella ◽  
...  

Abstract Background Implementation of guideline-based care for diabetes-related foot ulcers (DFU) in clinical practice is typically sub-optimal. We aimed to evaluate the cost-effectiveness of implementing different incremental increases in guideline-based care scenarios, compared with current practice, using discrete event simulation models. Methods The costs and effectiveness of current practice (identified as 30% receiving guideline-based care, remainder (70%) non-guideline-based care) were compared with seven hypothetical scenarios of implementing incremental increases in guideline-based care (40%, 50%, 60%, 70%, 80%, 90%, 100%) and evaluated using discrete event simulation models, including important events of disease history and parameterised by a large DFU cohort. Incremental cost-effectiveness ratio for each scenario was calculated and compared to willingness-to-pay of AUD28,000 per quality-adjusted life-years (QALY). Probability sensitivity analysis was conducted to incorporate parameter uncertainty by 2,000 random simulations. Results Under a three-year time-horizon, the seven scenarios (40%-100% guideline-based care) were estimated to incrementally save $901-$1843 (AUD 2020) and provide 0.017-0.056 more QALY per person, with all scenarios being cost-saving & more effective than current practice (30% guideline-based care). From probability sensitivity analyses we were increasingly confident (69.7%-89.8% confident) that implementing increased incremental scenarios (40%-100% guideline-based care) would be cost-effective compared with current practice (59.8%-73.4% confident). Conclusions All scenarios incrementally increasing guideline-based care were evaluated to be cost-saving and more effective than current practice according to this discrete event simulation modelling based on a large real-world cohort. Key messages Our findings support the cost-effectiveness of implementing any incremental increase in guideline-based care compared to current practice.


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