A Modeling Study of the Cost-Effectiveness of a Risk-Stratified Surveillance Program for Melanoma in the United Kingdom

Background

Population-wide screening for melanoma is unlikely to be cost-effective. Nevertheless, targeted surveillance of high-risk individuals may be.

Cost-Effectiveness of Disease-Modifying Therapies in the Management of Multiple Sclerosis for the Medicare Population

Objective:  To evaluate the cost-effectiveness of disease-modifying therapies (DMTs) for the management of multiple sclerosis (MS) compared to best supportive care in the United States.
Methods:  Cost-effectiveness analysis was undertaken using a state transition model of disease natural history and the impact of DMTs for the representative Medicare beneficiary with MS. Costs and outcomes were evaluated from the health-care payer perspective using a 50-year time horizon. Natural history data were drawn from a longitudinal cohort study. The effectiveness of the DMTs was evaluated through a systematic review. Utility data were taken from a study of patients with clinically definite MS in Nova Scotia. Resource use and cost data were derived from the Sonya Slifka database and associated literature.
Results:  When based on placebo-controlled evidence, the marginal cost-effectiveness of interferon beta (IFNβ) and glatiramer acetate compared to best supportive care is expected to be in excess of $100,000 per quality-adjusted life-year gained. When evidence from head-to-head trials is incorporated into the model, the cost-effectiveness of 6 MIU IFNβ-1a is expected to be considerably less favorable. Treatment discontinuation upon progression to Expanded Disability Status Scale 7.0 is expected to improve the cost-effectiveness of all DMTs.
Conclusions:  Further research is required to examine the long-term clinical effectiveness and cost-effectiveness of these therapies. There is no definitive guidance in the United States concerning discontinuation of DMTs; this study suggests that the prudent use of a treatment discontinuation rule may considerably improve the cost-effectiveness of DMTs.     

肝硬化患者肝功能检验临床分析

目的研究肝硬化中肝功能检验的应用价值。方法选择我院2016年10月-2019年10月接诊的肝硬化病患60例为试验组,选择我院同期接诊的健康体检者60例作为对照组。2组都接受肝功能检验,包括总胆汁酸(total bile acid,TBA)、白蛋白(albumin,ALB)、血清胆碱酯酶(serum cholinesterase,CHE)和血清胆固醇(serum cholesterol,CHO),并对其检验结果做出对比。结果试验组TBA水平比对照组高,P<0.05。试验组CHE、ALB和CHO水平比对照组低,P<0.05。对于肝功能分级为A级、B级和C级的病患来说,其CHE、ALB、TBA和CHO水平比较差异有统计学意义,P<0.05。结论积极开展肝功能检验工作,能够为肝硬化的诊断和等级评估提供重要指导。     

Cost-Effectiveness of Chemotherapy for Breast Cancer and Age Effect in Older Women

BackgroundPrevious economic evaluations compared specific chemotherapy agents using input parameters from clinical trials and resource utilization costs. Cost-effectiveness of treatment groups (drug classes) using community-level effectiveness and cost data, however, has not been assessed for elderly patients with breast cancer.ObjectiveTo assess the cost-effectiveness of chemotherapy regimens by age and disease stage under “real-world” conditions for patients with breast cancer.MethodsThe Surveillance Epidemiology and End Results-Medicare data were used to identify patients with breast cancer with American Joint Committee on Cancer stage I/II/IIIa, hormone receptor–negative (estrogen receptor–negative and progesterone receptor–negative) patients from 1992 to 2009. Patients were categorized into three adjuvant treatment groups: 1) no chemotherapy, 2) anthracycline, and 3) non–anthracycline-based chemotherapy. Median life-years and quality-adjusted life-years (QALYs) were measured using Kaplan-Meier analysis and were evaluated against average total health care costs (2013 US dollars).ResultsA total of 4575 patients (propensity score–matched) were included for the primary analysis. The anthracycline group experienced 12.05 QALYs and mean total health care costs of $119,055, resulting in an incremental cost-effectiveness ratio of $7,688 per QALY gained as compared with the no chemotherapy group (QALYs 7.81; average health care cost $86,383). The non–anthracycline-based group was dominated by the anthracycline group with lower QALYs (9.56) and higher health care costs ($122,791). Base-case results were found to be consistent with the best-case and worst-case scenarios for utility assignments. Incremental cost-effectiveness ratios varied by age group (range $3,790–$90,405 per QALY gained).ConclusionsAnthracycline-based chemotherapy was found cost-effective for elderly patients with early stage (stage I, II, IIIa) breast cancer considering the US threshold of $100,000 per QALY. Further research may be needed to characterize differential effects across age groups.     

基于剪切波的肝脏纤维化超声粘弹性检测系统

描述了一种基于目前医用超声硬件的肝脏粘弹性测试系统。该系统利用超声辐射力推动肝脏软组织形成瞬态响应,探测该响应中的剪切波传播,从而估算肝脏组织的弹性和粘性。本研究证实肝脏的杨氏模量和粘性系数可以通过剪切波成功定量检测,并且可以用目前已有的医用超声系统实现而无需额外硬件,从而为医生提供一种新的廉价而有效便捷的诊断手段。     

Cost-Effectiveness of Sorafenib for Second-Line Treatment of Advanced Renal Cell Carcinoma

Objectives:  To estimate the cost-effectiveness of sorafenib (Nexavar, Bayer, Leverkusen, Germany) versus best supportive care (BSC) for second-line treatment of advanced renal cell carcinoma from the perspective of the UK National Health Service.
Methods:  A decision analytic model was developed to estimate the cost-effectiveness of sorafenib. The clinical effectiveness of sorafenib versus BSC was taken from a recent randomized phase III trial. Utility values were taken from a phase II trial of sunitinib, using EQ-5D tariffs. Cost data were obtained from published literature and were based on current UK practice. The effect of parameter uncertainty on cost-effectiveness was explored through extensive one-way and probabilistic sensitivity analyses.
Results:  Compared to BSC, sorafenib treatment resulted in an incremental cost per quality-adjusted life year (QALY) gained of £75,398, based on an estimated mean gain of 0.27 QALYs per patient, at a mean additional cost of £20,063 (inflated to 2007/2008). The probability that sorafenib is cost-effective compared to BSC at a willingness to pay threshold of £30,000 per QALY is 0.0%. In sensitivity analysis, estimates of cost per QALY were sensitive to changes in the clinical effectiveness parameters, and to health state utilities and drug costs.
Conclusions:  Sorafenib has been shown to be clinically effective compared to BSC, offering additional health benefits; however, with a cost per QALY in excess of £70,000, it may not be regarded as a cost-effective use of resources in some health-care settings.     

Cost-Effectiveness of Repetitive Transcranial Magnetic Stimulation versus Antidepressant Therapy for Treatment-Resistant Depression

BackgroundRepetitive transcranial magnetic stimulation (rTMS) therapy is a clinically safe, noninvasive, nonsystemic treatment for major depressive disorder.ObjectiveWe evaluated the cost-effectiveness of rTMS versus pharmacotherapy for the treatment of patients with major depressive disorder who have failed at least two adequate courses of antidepressant medications.MethodsA 3-year Markov microsimulation model with 2-monthly cycles was used to compare the costs and quality-adjusted life-years (QALYs) of rTMS and a mix of antidepressant medications (including selective serotonin reuptake inhibitors, serotonin and norepinephrine reuptake inhibitors, tricyclics, noradrenergic and specific serotonergic antidepressants, and monoamine oxidase inhibitors). The model synthesized data sourced from published literature, national cost reports, and expert opinions. Incremental cost-utility ratios were calculated, and uncertainty of the results was assessed using univariate and multivariate probabilistic sensitivity analyses.ResultsCompared with pharmacotherapy, rTMS is a dominant/cost-effective alternative for patients with treatment-resistant depressive disorder. The model predicted that QALYs gained with rTMS were higher than those gained with antidepressant medications (1.25 vs. 1.18 QALYs) while costs were slightly less (AU $31,003 vs. AU $31,190). In the Australian context, at the willingness-to-pay threshold of AU $50,000 per QALY gain, the probability that rTMS was cost-effective was 73%. Sensitivity analyses confirmed the superiority of rTMS in terms of value for money compared with antidepressant medications.ConclusionsAlthough both pharmacotherapy and rTMS are clinically effective treatments for major depressive disorder, rTMS is shown to outperform antidepressants in terms of cost-effectiveness for patients who have failed at least two adequate courses of antidepressant medications.     

Cost-Effectiveness of a Community-Level HIV Risk Reduction Intervention for Women Living in Low-Income Housing Developments

We conducted a cost-effectiveness analysis of a multi-site community-level HIV prevention trial that enrolled women living in 18 low-income housing developments in 5 U.S. cities. A mathematical model of HIV transmission was used to estimate the number of HIV infections averted and quality-adjusted life years (QALYs) saved by the community-level intervention, based on data obtained from community-wide sexual behavior surveys at baseline and 12-month follow-up. Results indicated that the intervention prevented approximately 1 infection per 3500 women reached by the intervention, at a total cost of $174,845. The cost per QALY saved by the intervention was $37,433 and the cost per HIV infection averted was $732,072. The community-level intervention was moderately cost-effective in comparison with other HIV prevention programs for at-risk women. Synergistic approaches to HIV prevention that combine community-level sexual norm change interventions with more intensive risk reduction programs for high-risk women are needed.Editors’ Strategic Implications:The authors present a promising and efficient community-level HIV prevention approach, with effects beyond the limited scope of individual or small group interventions. This paper represents an example of how an analysis of cost-effectiveness can provide policymakers with information needed for difficult decisions about prevention resource allocations.     

Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in The Netherlands

ObjectivesSpinal muscular atrophy (SMA) is a rare genetic disorder that causes progressive muscle weakness and paralysis. In its most common and severe form, the majority of untreated infants die before 2 years of age. Early detection and treatment, ideally before symptom onset, maximize survival and achievement of age-appropriate motor milestones, with potentially substantial impact on health-related quality of life. Therefore, SMA is an ideal candidate for inclusion in newborn screening (NBS) programs. We evaluated the cost-effectiveness of including SMA in the NBS program in The Netherlands.MethodsWe developed a cost-utility model to estimate lifetime health effects and costs of NBS for SMA and subsequent treatment versus a treatment pathway without NBS (ie, diagnosis and treatment after presentation with overt symptoms). Model inputs were based on literature, local data, and expert opinion. Sensitivity and scenario analyses were conducted to assess model robustness and validity of results.ResultsAfter detection of SMA by NBS in 17 patients, the number of quality-adjusted life-years gained per annual birth cohort was estimated at 320 with NBS followed by treatment compared with treatment after clinical SMA diagnosis. Total healthcare costs, including screening, diagnostics, treatment, and other healthcare resource use, were estimated to be €12 014 949 lower for patients identified by NBS.ConclusionsNBS for early identification and treatment of SMA versus later symptomatic treatment after clinical diagnosis improves health outcomes and is less costly and, therefore, is a cost-effective use of resources. Results were robust in sensitivity and scenario analyses.     

A Systematic Review and Narrative Synthesis of Health Economic Evaluations of Hepatocellular Carcinoma Screening Strategies

ObjectivesMany economic evaluations of hepatocellular carcinoma (HCC) screenings have been conducted; however, these vary substantially with regards to screening strategies, patient group, and setting. This review aims to report the current knowledge of the cost-effectiveness of screening and describe the published data.MethodsWe conducted a search of biomedical and health economic databases up to July 2020. We included full and partial health economic studies if they evaluated the costs or outcomes of HCC screening strategies.ResultsThe review included 43 studies. Due to significant heterogeneity in key aspects across the studies, a narrative synthesis was conducted. Most studies reported using ultrasound or alpha fetoprotein as screening strategies. Screening intervals were mostly annual or biannual. Incidence, diagnostic performance, and health state utility values were the most critical parameters affecting the cost-effectiveness of screening. The majority of studies reported HCC screening to be cost-effective, with the biannual ultrasound + alpha fetoprotein standing out as the most cost-effective strategy. However, few studies considered the utilization rate, and none considered the diagnostic performance of ultrasound in the context of central adiposity. Computed tomography and magnetic resonance imaging were also evaluated, but its cost-effectiveness was still controversial.ConclusionsAlthough many studies suggested HCC screening was cost-effective, substantial limitations of the quality of these studies means the results should be interpreted with caution. Future modeling studies should consider the impact of central adiposity on the precision of ultrasound, real-world utilization rates and projections of increased HCC incidence.     

The Impact of Fewer Hip Fractures with Risedronate Versus Alendronate in the First Year of Treatment: Modeled German Cost-Effectiveness Analysis

Background:  The Risedronate and Alendronate (REAL) cohort study provides unique comparative effectiveness data for real world bisphosphonate treatment of osteoporosis.
Objective:  The objective of this analysis was to assess the cost-effectiveness of risedronate compared to generic alendronate in Germany applying the REAL effectiveness data.
Materials and Methods:  A validated Markov model of osteoporosis was populated with REAL effectiveness data and German epidemiological, cost, and utility data. To estimate the impact of therapy on hip fractures, costs, and quality adjusted life years (QALYs), the analysis included women ≥65 years, treated with risedronate or alendronate and followed for 4 additional years. Country-specific data included population mortality, fracture costs, and annual drug costs, using a German social insurance perspective. Costs and outcomes were discounted at 3%. A differential hip fracture relative risk reduction of 43% was applied to risedronate vs. alendronate.
Results:  The model predicted that treatment with risedronate would result in fewer hip fractures and more QALYs at a reduced cost (savings of €278 per treated woman) compared to treatment with generic alendronate. Sensitivity analysis assuming 2 years of treatment and equivalence of effect after 1 year show cost savings as well (€106 per treated woman).
Discussion:  Whereas previous economic evaluations involving bisphosphonates have mainly relied on efficacy data from noncomparative clinical trials, this study's strength is in the use of comparative effectiveness data from one data source. The magnitude of the cost savings observed were sensitive to alternative assumptions regarding treatment duration, therapy discontinuation and cost of generic alendronate.
Conclusions:  Based on "real world" data the analysis supports the first line use of risedronate for the treatment of osteoporotic women in Germany.     

Critical Analysis of Markov Models Used for the Economic Evaluation of Colorectal Cancer Screening: A Systematic Review

Background

The economic evaluation of colorectal cancer screening is challenging because of the need to model the underlying unobservable natural history of the disease.

Cost-Effectiveness of Using Clinical Risk Factors with and without DXA for Osteoporosis Screening in Postmenopausal Women

Background:  According to several guidelines, the assessment of postmenopausal fracture risk should be based on clinical risk factors (CRFs) and bone density. Because measurement of bone density by dual x-ray absorptiometry (DXA) is quite expensive, there has been increasing interest to estimate fracture risk by CRFs.
Objective:  The aim of this study was to determine the cost-effectiveness of osteoporosis screening of CRFs with and without DXA compared with no screening in postmenopausal women in Germany.
Methods:  A cost-utility analysis and a budget-impact analysis were performed from the perspective of the statutory health insurance. A Markov model simulated costs and benefits discounted at 3% over lifetime.
Results:  Cost-effectiveness of CRFs compared with no screening is €4607, €21,181, and €10,171 per quality-adjusted life-year (QALY) for 60-, 70-, and 80-year-old women, respectively. Cost-effectiveness of DXA plus CRFs compared with CRFs alone is €20,235 for 60-year-old women. In women above the age of 70, DXA plus CRFs dominates CRFs alone. DXA plus CRFs results in annual costs of €175 million, or 0.4% of the statutory health insurance's annual budget.
Conclusion:  Funders should be careful in adopting a strategy based on CRFs alone instead of DXA plus CRFs. Only if DXA is not available, assessing CRFs only is an acceptable option in predicting a woman's risk of fracture.     

Cost-Utility Analysis of a Complex Intervention to Reduce School-Based Bullying and Aggression: An Analysis of the Inclusive RCT

ObjectivesBullying and aggression among children and young people are key public mental health priorities. In this study, we evaluated the cost-effectiveness of a complex school-based intervention to address these outcomes within a large-cluster randomized trial (Inclusive).MethodsForty state secondary schools were randomly allocated (1:1) to receive the intervention or continue with current practice as controls. Data were collected using paper questionnaires completed in classrooms including measures of their health-related quality of life using the Childhood Utility Index and police and National Health Service resource use. Further detailed data were collected on the cost of delivering the intervention. We calculated incremental cost-effectiveness ratios following the intention-to-treat principle using multilevel linear regression models that allowed for clustering of pupils at the school level.ResultsOverall, we found that the intervention was highly cost-effective, with cost-per quality-adjusted life year thresholds of £13 284 and £1875 at 2 years and 3 years, respectively. Analysis of uncertainty in the result at 2 years revealed a 65% chance of being cost-effective, but after 3 years there was a 90% chance that it was cost-effective.ConclusionThis study provides strong evidence collected prospectively from a randomized study that this school-based intervention is highly cost-effective. Education- and health-sector policy makers should consider investment in scaling up this intervention.     

Optimizing the Position and Use of Omalizumab for Severe Persistent Allergic Asthma Using Cost-Effectiveness Analysis

BackgroundThere has been some controversy on whether the costs of omalizumab outweigh its benefits for severe persistent allergic asthma.ObjectivesThis study aimed to resolve the uncertainties and limitations of previous analyses and establish the cost-effectiveness of omalizumab under the list price and Patient Access Scheme (PAS) discounted price for the UK National Health Service.MethodsA decision-analytic model was developed to evaluate the long-term cost-effectiveness of omalizumab under the perspective of the National Health Service. Outcomes were expressed as quality-adjusted life-years (QALYs). Patient subgroups were defined post hoc on the basis of data collected in clinical trials: previous hospitalization, on maintenance oral corticosteroids, and three or more previous exacerbations.ResultsThe incremental cost-effectiveness ratio varied from £30,109 to £57,557 per QALY gained depending on the population considered using the PAS price; incremental cost-effectiveness ratios were over a third higher using the list price. Omalizumab is likely to be cost-effective at the threshold of £30,000 per QALY gained in the severe subgroups if the improvement in health-related quality of life from omalizumab is mapped from an asthma-specific measure to the EuroQol five-dimensional questionnaire (vs. the EuroQol five-dimensional questionnaire directly collected from patients) or asthma mortality refers to death after hospitalization from asthma (vs. asthma-mortality risk in the community).ConclusionsAlthough the cost-effectiveness of omalizumab is more favorable under the PAS price, it represents good value for money only in severe subgroups and under optimistic assumptions regarding asthma mortality and improvement in health-related quality of life. For these reasons, omalizumab should be carefully targeted to ensure value for money.     

The Cost-Effectiveness of Different Hearing Screening Strategies for 50- to 70-Year-Old Adults: A Markov Model

ObjectiveTo assess the cost-effectiveness of screening 50- to 70-year-old adults for hearing loss in The Netherlands. We compared no screening, telephone screening, Internet screening, screening with a handheld screening device, and audiometric screening for various starting ages and a varying number of repeated screenings.MethodsThe costs per quality-adjusted life-year (QALY) for no screening and for 76 screening strategies were analyzed using a Markov model with cohort simulation for the year 2011. Screening was deemed to be cost-effective if the costs were less than €20,000/QALY.ResultsScreening with a handheld screening device and audiometric screening were generally more costly but less effective than telephone and Internet screening. Internet screening strategies were slightly better than telephone screening strategies. Internet screening at age 50 years, repeated at ages 55, 60, 65, and 70 years, was the most cost-effective strategy, costing €3699/QALY. At a threshold of €20,000/QALY, this strategy was with 100% certainty cost-effective compared with current practice and with 69% certainty the most cost-effective strategy among all strategies.ConclusionsThis study suggests that Internet screening at age 50 years, repeated at ages 55, 60, 65, and 70 years, is the optimal strategy to screen for hearing loss and might be considered for nationwide implementation.     

Cost-Effectiveness of Telaprevir in Patients with Genotype 1 Hepatitis C in Australia

BackgroundProtease inhibitors such as telaprevir (Incivo) are reimbursed in Australia for the treatment of patients with genotype 1 hepatitis C virus (HCV) infection in combination with pegylated interferon and ribavirin (PR).ObjectivesTo assess the cost-effectiveness of telaprevir plus PR compared with PR alone in 1) previously untreated patients and 2) patients who had received treatment with PR earlier.MethodsSustained virological response rates and average treatment durations of telaprevir and PR (given with or without telaprevir) were taken from the telaprevir ADVANCE and REALIZE clinical trials but were modified to take account of differences in prescribing rules between the trials and Australian clinical practice. The probability of transitioning between Markov disease states was based on data from the Australian Kirby Institute where possible and supplemented using data from the published literature. Utility values obtained from the EuroQol five-dimensional questionnaire data collected in the ADVANCE and REALIZE trials were used to represent the utility during HCV treatment. Utility values for Markov health states were taken from the published literature. Unit costs (2014 AU $) were taken from Australian sources.ResultsIn treatment-naive patients, the discounted cost per life-years gained was AU $37,706 and the discounted cost per quality-adjusted life-year was AU $19,283. In treatment-experienced patients, the discounted cost per life-year gained was AU $23,855 and the discounted cost per quality-adjusted life-year was AU $14,948.ConclusionTelaprevir plus PR in the Australian setting is cost-effective when compared with PR alone in patients infected with genotype 1 HCV.     

Cost-Effectiveness of Drug-Eluting Stents in a US Medicare Setting: A Cost-Utility Analysis with 3-Year Clinical Follow-Up Data

Background:  There is only limited information about cost-effectiveness of drug-eluting compared with bare metal stents (BMS) over a time horizon of more than 1 year.
Methods and Results:  We developed a Markov model based on clinical outcome data from a meta-analysis including 17 randomized controlled trials comparing drug-eluting versus BMS with a minimum follow-up of 1 (n = 8221) and a maximum follow-up of 3 years (n = 4105) in patients with chronic coronary artery disease. Costs were obtained as reimbursement rates for diagnosis related groups from the US Centers for Medicare and Medicaid Services. All costs and effects were discounted at 3% annually. All costs are reported in US dollars of the financial year 2007. The incremental effects are 0.002 (95% confidence interval −0.039 to 0.041) quality-adjusted life-years (QALYs) for the sirolimus- and −0.001 (−0.040 to 0.038) QALYs for the paclitaxel-eluting stents (PES). The incremental costs are $2790 for the sirolimus- and $3838 for the PES. The incremental cost-effectiveness ratio is >$1,000,000 per QALY for the sirolimus-eluting stent. PES are dominated by BMS (i.e., less effective and more costly). Among various sensitivity analyses performed, the model proved to be robust.
Conclusions:  Our analysis from a US Medicare perspective suggests that drug-eluting stents are not cost-effective compared with BMS when implanted in unselected patients with symptomatic ischemic coronary artery disease.