scholarly journals Comparing Methods for Handling Missing Cost and Outcome Data in Clinical Trial-based Cost-effectiveness Analysis

2021 ◽  
Author(s):  
Modou Diop ◽  
David Epstein
Keyword(s):  
Cost Effectiveness ◽  
Repeated Measures ◽  
Cost Effectiveness Analysis ◽  
Sensitivity Analyses ◽  
Venous Ulceration ◽  
Time Horizons ◽  
Effectiveness Analysis ◽  
Life Years ◽  
Significant Difference ◽  
Endovenous Ablation

Abstract OBJECTIVES: This study compares methods for handling missing data to conduct cost-effectiveness analysis in the context of a clinical study.METHODS: Patients in the Early Endovenous Ablation in Venous Ulceration (EVRA) trial had between 1 year and 5.5 years (median 3 years) of follow-up under early or deferred endovenous ablation. This study compares Complete-Case-Analysis (CCA), multiple imputation using linear regression (MILR) and using predictive mean matching (MIPMM), Bayesian parametric approach using the R package missingHE (BPA) and repeated measures mixed model (RMM). The outcomes were total mean costs and total mean quality-adjusted life years (QALYs) at different time horizons (1 year, 3 years and 5 years). RESULTS: All methods found no statistically significant difference in cost at the 5% level in all time horizons, and all methods found statistically significantly greater mean QALY at year 1. By year 3, only BPA showed a statistically significant difference in QALY. Standard errors differed substantially between the methods employed. CONCLUSION: CCA can be biased if data are MAR, and is wasteful of the data. Hence the results for CCA are likely to be inaccurate. Other methods coincide in suggesting that early intervention is cost-effective at a threshold of £20,000 per QALY over all time horizons. However, the variation in the results across the methods does generate some additional methodological uncertainty, underlining the importance of conducting sensitivity analyses using alternative approaches.

scholarly journals Comparing Methods for Handling Missing Cost and Outcome Data in Clinical Trial-based Cost-effectiveness Analysis

2021 ◽  
Author(s):  
Modou Diop ◽  
David Epstein
Keyword(s):  
Clinical Trial ◽  
Cost Effectiveness ◽  
Repeated Measures ◽  
Cost Effectiveness Analysis ◽  
Simulation Studies ◽  
Time Horizons ◽  
Effectiveness Analysis ◽  
Life Years ◽  
Significant Difference ◽  
Better Than

Abstract OBJECTIVES: This study compares methods for handling missing data to conduct cost-effectiveness analysis in the context of a clinical study.METHODS: A long-term clinical trial with staggered recruitment was used as a case study (EVRA, NIHR-HTA project 11/129/197). Patients had between 1 year and 5.5 years (median 3 years) of follow-up under “early” or “deferred” treatment. The methods compared were Complete-Case-Analysis (CCA), multiple imputation using linear regression (MILR) and using predictive mean matching (MIPMM), Bayesian parametric approach using the R package missingHE (BPA) and repeated measures mixed model (RMM). The outcomes were total mean costs and total mean quality-adjusted life years (QALYs) at different time horizons (1 year, 3 years and 5 years). RESULTS: All methods found no statistically significant difference in cost at the 5% level in all years, and all methods found statistically significantly greater mean QALY at year 1. At year 3, BPA showed a statistically significant difference while other method did not at later time horizons. Standard errors differed substantially between the methods employed. CONCLUSION: MIPMM seemed to perform better than MILM, confirming findings from simulation studies. RMM and BPA might be feasible options, though they did not perform better than MIPMM in this dataset. Further simulation studies and applications should continue to compare these methods.

Ixazomib (IXA), carfilzomib (CAR), elotuzumab (ELO) or daratumumab (DAR) with lenalidomide and dexamethasone (LEN+DEX) versus LEN+DEX only in relapsed/refractory multiple myeloma (R/R MM): A comparative cost-effectiveness analysis.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 8043-8043
Author(s):  
Mavis Obeng-Kusi ◽  
Daniel Arku ◽  
Neda Alrawashdh ◽  
Briana Choi ◽  
Nimer S. Alkhatib ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Cost Effective ◽  
Cost Utility ◽  
Cost Effectiveness Analysis ◽  
The Other ◽  
Sensitivity Analyses ◽  
Treatment Comparison ◽  
Comparative Cost ◽  
Effectiveness Analysis ◽  
Life Years

8043 Background: IXA, CAR, ELO and DARin combination with LEN+DEXhave been found superior in efficacy compared to LEN+DEX in the management of R/R MM. Applying indirect treatment comparisons from a network meta-analysis (NMA), this economic evaluation aimed to estimate the comparative cost-effectiveness and cost-utility of these four triplet regimens in terms of progression-free survival (PFS). Methods: In the absence of direct treatment comparison from a single clinical trial, NMA was used to indirectly estimate the comparative PFS benefit of each regimen. A 2-state Markov model simulating the health outcomes and costs was used to evaluate PFS life years (LY) and quality-adjusted life years (QALY) with the triplet regimens over LEN+DEX and expressed as the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR). Probability sensitivity analyses were conducted to assess the influence of parameter uncertainty on the model. Results: The NMA revealed that DAR+LEN+DEX was superior to the other triplet therapies, which did not differ statistically amongst them. As detailed in the Table, in our cost-effectiveness analysis, all 4 triplet regimens were associated with increased PFSLY and PFSQALY gained (g) over LEN+DEX at an additional cost. DAR+LEN+DEX emerged the most cost-effective with ICER and ICUR of $667,652/PFSLYg and $813,322/PFSQALYg, respectively. The highest probability of cost-effectiveness occurred at a willingness-to-pay threshold of $1,040,000/QALYg. Conclusions: Our economic analysis shows that all the triplet regimens were more expensive than LEN +DEX only but were also more effective with respect to PFSLY and PFSQALY gained. Relative to the other regimens, the daratumumab regimen was the most cost-effective.[Table: see text]

scholarly journals Cost-effectiveness analysis of screening for first-degree relatives of patients with bicuspid aortic valve

2021 ◽  
Vol 42 (Supplement_1) ◽  
Author(s):  
I Tessler ◽  
M Leshno ◽  
A Shmueli ◽  
S Shpitzen ◽  
R Durst ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Aortic Valve ◽  
Bicuspid Aortic Valve ◽  
Cost Effectiveness Analysis ◽  
Public Institution ◽  
Sensitivity Analyses ◽  
Decision Analytic Model ◽  
First Degree Relatives ◽  
Effectiveness Analysis ◽  
Life Years

Abstract Introduction Bicuspid aortic valve (BAV) is the commonest congenital heart valve defect, found in 1% to 2% of the general population and associated with life-threatening complications. Given the high heritability index of BAV, many experts recommend echocardiography for first-degree relatives (FDRs) of an index patient. However, the cost-effectiveness of such cascade screening for BAV has not been fully evaluated. Materials and methods Using a decision-analytic model, we performed a cost-effectiveness analysis of echocardiographic screening of FDRs of BAV index cases. Data on BAV probabilities and BAV complications among FDRs were derived from our institution's BAV familial cohort and from the relevant literature on population-based BAV cohorts with long-term follow-up. Health gain was measured as quality-adjusted life years (QALYs). Cost inputs were based on list prices and literature data. One-way and probabilistic sensitivity analyses were performed to account for uncertainty in the model's variables. Results and disscusion Screening of FDRs was found to be the dominant strategy, being more effective and less costly than no screening, with savings of €208 and gains of 1.6 QALYs. Results were sensitive to the full range of reported BAV rates among FDRs across the literature, with the benefit gradually decreasing from the screening age of 55 years, with trend shifting at the age of 69. Conclusions This economic evaluation model revealed that echocardiographic screening of FDRs of BAV index case is not only clinically important but also highly cost effective and cost-saving. Health gains could be achieved from initiating screening program, along with costs saving. Sensitivity analysis supported the model's robustness, suggesting its generalization. FUNDunding Acknowledgement Type of funding sources: Public Institution(s). Main funding source(s): Center for Interdisciplinary Data Science Research fellowships grant

scholarly journals Comparing Prednisone and Methotrexate to Off-label Biologic Infliximab for Management of Ocular Uveitis: A Cost-minimization Analysis

10.36469/9895 ◽  
2015 ◽  
Vol 2 (2) ◽  
pp. 131-146
Author(s):  
William V. Padula ◽  
Miguel Cordero-Coma ◽  
Taygan Yilmaz ◽  
William V. Padula ◽  
Michéal J. Gallagher ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
The United States ◽  
Posterior Uveitis ◽  
Cost Effectiveness Analysis ◽  
Sensitivity Analyses ◽  
Off Label ◽  
Effectiveness Analysis ◽  
Life Years ◽  
Successful Recovery

Background: Approximately 3.75% of cases of blindness in the United States are caused by uveitis. Incurred clinical costs and lost productivity related to vision loss in these cases totals $3.58 billion annually. Objective: To evaluate whether infliximab, a modern off-label biologic, is cost-effective for treating posterior uveitis and panuveitis compared to current standards of care, methotrexate and prednisone. Methods: A cost-effectiveness analysis using a Markov model to simulate a patient cohort with posterior uveitis or panuveitis. The model followed patients’ therapy from the onset of posterior uveitis or panuveitis using the U.S. societal perspective. The lifetime model simulated health states that could lead to successful reversal of uveitis with standard or intensified treatment with prednisone, methotrexate, or infliximab. Probabilities, health utilities, and costs were included in the model based on findings from the literature. We conducted univariate sensitivity analyses and a Bayesian multivariate probablistic sensitivity analysis to estimate uncertainty in results. Outcomes were measured in terms of costs ($US, 2010) and effects (qualityadjusted life years; QALYs) discounted at 3% per year were estimated for each simulated treatment. An incremental cost-effectiveness ratio (ICER) for pairwise results was interpretted assuming a predetermined willingness-to-pay threshold of $100,000/QALY. Results: Average lifetime costs and QALYs for each drug were ($306.95; 15.80 QALYs) for prednisone, methotrexate ($36,232.24; 16.21 QALYs), and inflixmab ($74,762.63; 15.04 QALYs). Methotrexate was on average compared to prednisone, with an ICER of $86,901.16/QALY. Prednisone and methotrexate dominated infliximab. Sensitivity analyses suggested that the model was most sensitive to the utility for successful recovery from uveitis. The probabilistic sensitivity analysis returned results similar to the base case. Conclusion: This cost-effectiveness analysis suggests that despite advances in the use of biologics for treating sight-threatening posterior uveitis and panuveitis, infliximab had lower effectiveness and higher costs compared to both prednisone and methotrexate. As compared to prednisone, methotrexate was associated with increased costs and QALYs and was found to be a good value. Clinical trials of infliximab in the uveitis population are needed to reduce the uncertain estimates of inflixmab treatment success and the drug’s cost-effectiveness.

scholarly journals Cost-effectiveness analysis of Vaborem in Carbapenem-resistant Enterobacterales (CRE) -Klebsiella pneumoniae infections in Italy

2021 ◽  
Vol 11 (1) ◽  
Author(s):  
Francesco Saverio Mennini ◽  
Mario Gori ◽  
Ioanna Vlachaki ◽  
Francesca Fiorentino ◽  
Paola La Malfa ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Klebsiella Pneumoniae ◽  
Incremental Cost ◽  
Cost Effective ◽  
Cost Effectiveness Analysis ◽  
Sensitivity Analyses ◽  
Fixed Dose ◽  
Carbapenem Resistant ◽  
Effectiveness Analysis ◽  
Life Years

Abstract Background Vaborem is a fixed dose combination of vaborbactam and meropenem with potent activity against target Carbapenem-resistant Enterobacterales (CRE) pathogens, optimally developed for Klebsiella pneumoniae carbapenemase (KPC). The study aims to evaluate the cost-effectiveness of Vaborem versus best available therapy (BAT) for the treatment of patients with CRE-KPC associated infections in the Italian setting. Methods A cost-effectiveness analysis was conducted based on a decision tree model that simulates the clinical pathway followed by physicians treating patients with a confirmed CRE-KPC infection in a 5-year time horizon. The Italian National Health System perspective was adopted with a 3% discount rate. The clinical inputs were mostly sourced from the phase 3, randomised, clinical trial (TANGO II). Unit costs were retrieved from the Italian official drug pricing list and legislation, while patient resource use was validated by a national expert. Model outcomes included life years (LYs) and quality adjusted life years (QALYs) gained, incremental costs, incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR). Deterministic and probabilistic sensitivity analyses were also performed. Results Vaborem is expected to decrease the burden associated with treatment failure and reduce the need for chronic renal replacement therapy while costs related to drug acquisition and long-term care (due to higher survival) may increase. Treatment with Vaborem versus BAT leads to a gain of 0.475 LYs, 0.384 QALYs, and incremental costs of €3549, resulting in an ICER and ICUR of €7473/LY and €9246/QALY, respectively. Sensitivity analyses proved the robustness of the model and also revealed that the probability of Vaborem being cost-effective reaches 90% when willingness to pay is €15,850/QALY. Conclusions In the Italian setting, the introduction of Vaborem will lead to a substantial increase in the quality of life together with a minimal cost impact, therefore Vaborem is expected to be a cost-effective strategy compared to BAT.

Ulcus Cruris – Frühe venöse Ablation erhöht Lebensqualität bei Kosteneffizienz

2019 ◽  
Vol 24 (05) ◽  
pp. 220-220
Author(s):  
Gabriele Dobler
Keyword(s):  
Clinical Trial ◽  
Cost Effectiveness ◽  
Randomized Clinical Trial ◽  
Ulcus Cruris ◽  
Cost Effectiveness Analysis ◽  
Venous Reflux ◽  
Venous Ulceration ◽  
Effectiveness Analysis ◽  
Endovenous Ablation

Epstein DM. et al. Cost-effectiveness analysis of a randomized clinical trial of early versus deferred endovenous ablation of superficial venous reflux in patients with venous ulceration. Br J Surg 2019; 106: 555–562 Aktuelle Richtlinien empfehlen bei venösen Beingeschwüren die Behandlung des venösen Refluxes mit endovenösen Ablationstechniken. Viele Praktiker warten jedoch mit der Intervention bis das Ulcus abgeheilt ist. Die EVRA (Early Venous Reflux Ablation) Studie ergab, dass eine frühe endovenöse Ablation zu signifikant schnellerer Abheilung der Ulcera führte. Epstein et al. verglichen die Kosteneffektivität früher und später Intervention.

A Cost-Effectiveness Analysis of Rooming-in and Breastfeeding in Neonatal Opioid Withdrawal

2019 ◽  
Vol 37 (01) ◽  
pp. 001-007 ◽  
Author(s):  
Carmen M. Avram ◽  
Leah Yieh ◽  
Dmitry Dukhovny ◽  
Aaron B. Caughey
Keyword(s):  
Cost Effectiveness ◽  
The United States ◽  
Cost Effectiveness Analysis ◽  
Opioid Withdrawal ◽  
Sensitivity Analyses ◽  
Annual Number ◽  
Decision Analytic Model ◽  
Formula Milk ◽  
Effectiveness Analysis ◽  
Life Years

Abstract Objective Our cost-effectiveness analysis investigated rooming-in versus not rooming-in to determine optimal management of neonates with neonatal opioid withdrawal (NOW). Study Design A decision-analytic model was constructed using TreeAge to compare rooming-in versus not rooming-in in a theoretical cohort of 23,200 newborns, the estimated annual number affected by NOW in the United States. Additional considerations included the effect of breast milk versus formula milk in evaluating the need for pharmacotherapy. Primary outcomes were needed for pharmacotherapy and neurodevelopment. We assumed a societal perspective in evaluating costs and maternal-neonatal quality-adjusted life years (QALYs) using a willingness-to-pay threshold of $100,000/QALY. Model inputs were derived from literature and varied in sensitivity analyses. Results Rooming-in resulted in fewer neonates requiring pharmacotherapy when compared with not rooming-in. The rooming-in group had more neonates with intact/mild neurodevelopmental impairment and fewer cases of moderate to severe impairment. Rooming-in resulted in cost savings of $509,652,728 and 12,333 additional QALYs per annual cohort. When the risk ratio of need for pharmacotherapy in rooming-in was varied across a clinically plausible range, rooming-in remained the cost-effective strategy. Conclusion Maternal rooming-in with newborns affected by NOW leads to reduced costs and increased effectiveness. Management strategies should optimize nonpharmacological interventions as first-line treatment.

scholarly journals Strict lockdown versus flexible social distance strategy for COVID-19 disease: a cost-effectiveness analysis

2020 ◽  
Author(s):  
Ben W. Mol ◽  
Jonathan Karnon
Keyword(s):  
Cost Effectiveness ◽  
Mortality Rates ◽  
Cost Effectiveness Analysis ◽  
Sensitivity Analyses ◽  
Social Distancing ◽  
The Public ◽  
Financial Costs ◽  
Effectiveness Analysis ◽  
Life Years ◽  
Life Years Lost

Objectives To balance the costs and effects comparing a strict lockdown versus a flexible social distancing strategy for societies affected by Coronavirus-19 Disease (COVID-19). Design Cost-effectiveness analysis. Participants We used societal data and COVID-19 mortality rates from the public domain. Interventions The intervention was a strict lockdown strategy that has been followed by Denmark. Reference strategy was flexible social distancing policy as was applied by Sweden. We derived mortality rates from COVID-19 national statistics, assumed the expected life years lost from each COVID-19 death to be 11 years and calculated lost life years until 31st August 2020. Expected economic costs were derived from gross domestic productivity (GDP) statistics from each country's official statistics bureau and forecasted GDP. The incremental financial costs of the strict lockdown were calculated by comparing Sweden with Denmark using externally available market information. Calculations were projected per one million inhabitants. In sensitivity analyses we varied the total cost of the lockdown (range -50% to +100%). Main outcome measure Financial costs per life years saved. Results In Sweden, the number of people who died with COVID-19 was 577 per million inhabitants, resulting in an estimated 6,350 life years lost per million inhabitants. In Denmark, where a strict lockdown strategy was installed for months, the number of people dying with COVID-19 was on average 111 per million, resulting in an estimated 1,216 life years per million inhabitants lost. The incremental costs of strict lockdown to save one life year was US$ 137,285, and higher in most of the sensitivity analyses. Conclusions Comparisons of public health interventions for COVID-19 should take into account life years saved and not only lost lives. Strict lockdown costs more than US$ 130,000 per life year saved. As our all our assumptions were in favour of strict lockdown, a flexible social distancing policy in response to COVID19 is defendable.

scholarly journals Bayesian Regression Model for a Cost-Utility and Cost-Effectiveness Analysis Comparing Punch Grafting Versus Usual Care for the Treatment of Chronic Wounds

2020 ◽  
Vol 17 (11) ◽  
pp. 3823 ◽  
Author(s):  
Carmen Selva-Sevilla ◽  
Elena Conde-Montero ◽  
Manuel Gerónimo-Pardo
Keyword(s):  
Cost Effectiveness ◽  
Usual Care ◽  
Chronic Wounds ◽  
Cost Utility ◽  
Cost Effectiveness Analysis ◽  
Bayesian Regression ◽  
Sensitivity Analyses ◽  
System Perspective ◽  
Effectiveness Analysis ◽  
Life Years

Punch grafting is a traditional technique used to promote epithelialization of hard-to-heal wounds. The main purpose of this observational study was to conduct a cost-utility analysis (CUA) and a cost-effectiveness analysis (CEA) comparing punch grafting (n = 46) with usual care (n = 34) for the treatment of chronic wounds in an outpatient specialized wound clinic from a public healthcare system perspective (Spanish National Health system) with a three-month time horizon. CUA outcome was quality-adjusted life years (QALYs) calculated from EuroQoL-5D, whereas CEA outcome was wound-free period. One-way sensitivity analyses, extreme scenario analysis, and re-analysis by subgroups were conducted to fight against uncertainty. Bayesian regression models were built to explore whether differences between groups in costs, wound-free period, and QALYs could be explained by other variables different to treatment. As main results, punch grafting was associated with a reduction of 37% in costs compared to usual care, whereas mean incremental utility (0.02 ± 0.03 QALYs) and mean incremental effectiveness (7.18 ± 5.30 days free of wound) were favorable to punch grafting. All sensitivity analyses proved the robustness of our models. To conclude, punch grafting is the dominant alternative over usual care because it is cheaper and its utility and effectiveness are greater.

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