Estimating the cost-effectiveness of an intervention in a clinical trial when partial cost information is available: a Bayesian approach

2007 ◽  
Vol 17 (1) ◽  
pp. 67-81 ◽  
Author(s):  
Paul C. Lambert ◽  
Lucinda J. Billingham ◽  
Nicola J. Cooper ◽  
Alex J. Sutton ◽  
Keith R. Abrams
Keyword(s):  
Clinical Trial ◽  
Cost Effectiveness ◽  
Bayesian Approach ◽  
Cost Information ◽  
The Cost

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 ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Cost Effectiveness ◽  
Societal Perspective ◽  
Functional Disability ◽  
Health Benefit ◽  
Base Case ◽  
Current Standard ◽  
Disease Modifying Drugs ◽  
New Treatment ◽  
The Cost

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.

Cost-effectiveness of etanercept treatment in early active rheumatoid arthritis followed by dose adjustment

2011 ◽  
Vol 27 (3) ◽  
pp. 193-200 ◽  
Author(s):  
Gisela Kobelt ◽  
Ingrid Lekander ◽  
Andrea Lang ◽  
Bernd Raffeiner ◽  
Costantino Botsios ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Trial ◽  
Cost Effectiveness ◽  
Disease Activity ◽  
Dose Reduction ◽  
Societal Perspective ◽  
Active Rheumatoid Arthritis ◽  
Biologic Treatment ◽  
Early Ra ◽  
The Cost

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.

scholarly journals Cost-Effectiveness of Ipilimumab Plus Anti-PD-1 Therapy Versus Ipilimumab Alone in Patients With Metastatic Melanoma Resistant to Anti-PD-(L)1 Monotherapy

2021 ◽  
Vol 11 ◽  
Author(s):  
Ye Peng ◽  
Xiaohui Zeng ◽  
Liubao Peng ◽  
Qiao Liu ◽  
Lidan Yi ◽  
...  
Keyword(s):  
Cost Effectiveness ◽  
Metastatic Melanoma ◽  
Subgroup Analysis ◽  
Cost Effective ◽  
Quality Adjusted Life Year ◽  
Cost Information ◽  
Payer Perspective ◽  
The Us ◽  
The Cost ◽  
Quality Adjusted Life

ObjectiveThe use of ipilimumab plus anti-PD-1 has recently been shown to significantly improve the survival of patients with metastatic melanoma resistant to anti-PD-(L)1 monotherapy. The study assessed the cost-effectiveness of ipilimumab plus anti-PD-1 therapy in this population from the US payer perspective.Materials and MethodsA Markov model was created based on a retrospective analysis of patients with metastatic melanoma who were resistant to anti-PD-(L)1. Cost information was obtained from the Centers for Medicare and Medicaid Services and literature-based costs. The utility value was derived from the published literature. The results of the model was the total cost, quality-adjusted life-year (QALY), and incremental cost-effectiveness ratio (ICER). The uncertainty of the model was addressed through sensitivity analysis. In addition, we also conducted subgroup analysis.ResultsIpilimumab plus anti-PD-1 provided an improvement of 1.39 QALYs and 2.48 LYs, at a ICER of $73,163 per QALY. The HR of OS was the variable that had the greatest impact on ICER. Compared to ipilimumab, the probability of ipilimumab plus anti-PD-1 being cost-effective was 94% at the WTP of $150,000/QALY. The results of the subgroup analysis showed that the ICER in the majority of the subgroups was less than $150,000/QALY.ConclusionsIpilimumab plus anti-PD-1 was likely to be cost-effective compared to ipilimumab for patients with metastatic melanoma who are resistant to anti-PD-(L)1 at a WTP threshold of 150,000/QALY.

Design and Analytic Considerations in Determining the Cost-Effectiveness of Early Intervention in Asthma from a Multinational Clinical Trial

2001 ◽  
Vol 22 (4) ◽  
pp. 420-437 ◽  
Author(s):  
Sean D Sullivan ◽  
Bengt Liljas ◽  
Martin Buxton ◽  
Carl Johan Lamm ◽  
Paul O'Byrne ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Early Intervention ◽  
Cost Effectiveness ◽  
The Cost ◽  
Multinational Clinical Trial

Analysis of the Cost-Effectiveness of Mammography Promotion by Volunteers in Rural Communities

2002 ◽  
Vol 29 (6) ◽  
pp. 755-770 ◽  
Author(s):  
M. Robyn Andersen ◽  
Michelle Hager ◽  
Celina Su ◽  
Nicole Urban
Keyword(s):  
Cost Effectiveness ◽  
Rural Communities ◽  
Rural Areas ◽  
Cost Effective ◽  
Target Population ◽  
Individual Counseling ◽  
Cost Information ◽  
Community Activities ◽  
Community Volunteer ◽  
The Cost

The Community Trial of Breast Cancer Screening Promotion assessed the effectiveness and cost-effectiveness of mammography promotion by community volunteer groups in rural areas using three different intervention approaches: individual counseling, community activities, and a combined intervention including both. Societal costs of the interventions were calculated and used in conjunction with measures of effectiveness to calculate cost-effectiveness in terms of cost per additional mammogram and cost per year of life saved. Methods of collecting and using cost information to assess the cost-effectiveness of community interventions are described. The Community Activities intervention was found to be the most cost-effective, at approximately $2,000 for each additional regular mammography user in the community. The cost per year of life saved associated with mammography promotion was approximately $56,000 per year of life saved. Exploratory analyses suggest that the most cost-effective method of promoting mammography use may vary with the target population.

scholarly journals Economic Evaluation of Daratumumab and Pomalidomide and Dexamethasone Versus Isatuximab and Pomalidomide and Dexamethasone for Patients with Relapsed or Refractory Multiple Myeloma

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 19-20
Author(s):  
Neda Alrawashdh ◽  
Abdulaali Almutairi ◽  
Ali McBride ◽  
Ivo Abraham
Keyword(s):  
Clinical Trial ◽  
Multiple Myeloma ◽  
Cost Effectiveness ◽  
Time Horizon ◽  
Cost Utility ◽  
Phase Iii ◽  
Health States ◽  
Time Horizons ◽  
Life Years ◽  
The Cost

Background. Although several new treatments are available for patients with multiple myeloma (MM), most patients eventually relapse at a median time of 8.0 months (95%CI: 6.3-8.9). Patients with relapsed and refractory MM (R/R MM) who have had several lines of previous therapy or who are refractory to lenalidomide and proteasome inhibitors require alternative options. Daratumumab and isatuximab are monoclonal antibodies that bind to the human CD38 receptor. Phase II/III clinical trials showed that isatuximab (ISA) or daratumumab (DARA) in combination with pomalidomide (POM-d) and low-dose dexamethasone (DEXA) significantly improve progression-free survival (PFS) in patients with R/R MM. No studies have assessed the comparative efficacy and cost-effectiveness of both regimens in management of R/R MM. We performed an indirect comparison of both regimens in terms of PFS and overall survival (OS) and evaluated the cost-effectiveness and cost-utility of DARA+POM-d+DEXA and ISA+POM-d+DEXA from a US payer's perspective. Methods. A partitioned survival model was developed to create three health states (pre-progression, progression, and death). The model was run three times with different time horizons (one, three and five years). To simulate health outcomes for each treatment regimen, transition probabilities between the three health states were derived from parametric exponential and lognormal distributions fitted to Kaplan-Meier (KM) curves of PFS and OS of the phase Ib clinical trial (Chari et al.; Blood 2017) for DARA+POM-d+DEXA and the phase III clinical trial (Attal et al.; Lancet 2019) for ISA+POM-d+DEXA. Wholesale acquisition costs (WAC) were obtained from RedBook for each regimen. Pre-progression costs included costs of regimens; premedication (50 mg diphenhydramine, 650 mg acetaminophen, 50 mg ranitidine); managing side effects; routine care and monitoring; and medication administration. Costs were inflated based on the medical consumer price index to the second quarter of 2020. Utilities were obtained from literature and assumed the same for both interventions. Annual discount rate of 3.5% was applied for costs and outcomes beyond the first year. The life years (LY) and quality adjusted LY (QALY) for each treatment, and the incremental cost-effectiveness (ICER) and cost-utility ratios (ICUR) were estimated in both base and probabilistic sensitivity analyses (PSA:10,000 simulations). The cost-effectiveness plane (CEP) and cost-effectiveness acceptability curves (CEAC) were plotted. Results. In the naïve patient simulation, median PFS and OS were estimated to be 9.5 months and 18 months for DARA+POM-d+DEXA, and 14.5 months and 26 months for ISA+POM-d +DEXA. As shown in the table below, ISA+POM-d+DEXA is associated with greater LY and QALY gains at one-, three- and five-year time horizons. The costs of ISA+POM-d+DEXA at one- and three- year time horizons are less than that of DARA+POM-d+DEXA, which resulted in saving (decremental) ICERs. At 5 years' time horizon, ISA+POM-d+DEXA was associated with incremental benefits (0.57 LY, 0.35 QALY) and incremental costs of $88,271 when compared with DARA+POM-d+DEXA. Per the CEAC plot, the probability that ISA+POM-d+DEXA is cost-effective was 100%, 65% and 23% at a willingness to pay threshold (WTP) of $100,000 per QALY in one-, three- and five-year time horizons. Conclusions. Clinically, ISA+POM-d+DEXA is associated with incremental survival gains of ~1 month and quality-adjusted survival gains of 0.5 month than DARA+POM-d+DEXA when patients are treated for one year. The benefits increase with treatment duration to reach ~7 months life year gains and 4 months quality-adjusted life year gains if patients treated for 5 years. Due to its lower total costs, Isatuximab based-regimen yielded saving ICERs at one and three years. However, ISA+POM-d+DEXA cost exceeded DARA+POM-d+DEXA at 5 years' time horizon to yield an ICER above the WTP. Disclosures McBride: Merck: Speakers Bureau; Pfizer: Consultancy; Sandoz: Consultancy; MorphoSys: Consultancy; Bristol-Myers Squibb: Consultancy; Coherus BioSciences: Consultancy, Speakers Bureau. Abraham:Celgene: Consultancy; Terumo: Consultancy; Rockwell Medical: Consultancy; Janssen: Consultancy; Mylan: Consultancy; Sandoz: Consultancy; Coherus BioSciences: Research Funding, Speakers Bureau; MorphoSys: Consultancy.

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