应用Infopoll软件实现基于德尔菲法的医保预算影响分析数据获取

医保预算影响分析作为药物经济学评价的重要辅助手段,能有效衡量医保基金的可负担性,在医保准入谈判、医保报销目录调整和支付价格的制定过程中作用显著。医保预算影响分析的数据质量对分析结果影响较大,影响卫生决策的科学性,在现有数据无法满足要求的情况,需借助相关软件采用德尔菲法来保障数据准确性。Infopoll软件是一个功能强大、易于操作的应用软件,可通过提供多种问题选择和多形式的答案设置来制定专家咨询问卷,并提供详细的统计图表进行结果分析。本文采用实例剖析的方式,对应用该软件实现基于德尔菲法的医保预算影响分析数据获取进行简要介绍,并分析主要结果。     

Cost-effectiveness and Net Monetary Benefit of Durvalumab Consolidation Therapy Versus No Consolidation Therapy After Chemoradiotherapy in Stage III Non–small Cell Lung Cancer in the Italian National Health Service

PurposeThe aim of this study was to evaluate the cost-effectiveness and net monetary benefit of durvalumab consolidation therapy compared with no consolidation therapy after chemoradiotherapy in patients with stage III non–small cell lung cancer with programmed cell death 1 ligand 1 expression ≥1% from the Italian National Health Service perspective.MethodsWe developed a 12-month decision tree combined with a lifetime cohort Markov model in which patients were assigned to receive durvalumab consolidation therapy or active follow-up (Italian standard of care) after chemoradiotherapy to compare cost-effectiveness and net monetary benefit of the two strategies during a 40-year period. Clinical outcomes data were obtained from the respective clinical trials and extrapolated using survival analysis; cost data were derived from Italian official sources and relevant real-world studies. The incremental cost-effectiveness ratio, incremental cost-utility ratio, and incremental net monetary benefit were computed and compared against a 16,372 € per quality-adjusted life-year (QALY) willingness-to-pay threshold. We performed deterministic sensitivity analysis and probabilistic sensitivity analysis to assess how uncertainty affected results; we also performed scenario analyses to compare results under different pricing settings.FindingsIn the base-case scenario, during a 40-year period, the total costs for patients treated with durvalumab consolidation therapy and active follow-up were €59,860 and €49,840 respectively; life-years gained were 3.47 and 3.31, respectively; and QALYs gained were 2.73 and 2.50, respectively, with an incremental cost-effectiveness ratio of €62,131 per life-year, an incremental cost-utility ratio of €42,322 per QALY, and an incremental net monetary benefit of €−6,144. We found that durvalumab was cost-effective (incremental net monetary benefit = 0) when a discount of 13% and 30% on its official price was applied, considering all other drugs priced according to official or maximum selling prices, respectively. Results were most sensitive to the progression-free survival rate for durvalumab and active follow-up, health utility in progression-free state, and price of subsequent treatments.ImplicationsOur analysis indicates that durvalumab consolidation is cost-effective when a discount is applied on its official price. These results suggest that durvalumab may deliver an incremental health benefit with a contained upfront cost during a 40-year period, from the Italian National Health Service perspective, providing added value in a potentially curative care setting.     

Treatment of primary immunodeficiency with Kiovig®: a literature review

Introduction

Primary immunodeficiency disorders are associated with increased patient susceptibility to recurrent infections. Since the 1950s, immunoglobulin products have been administered to treat infections in primary immunodeficiency, and patients often require lifelong therapy. The aim of this study is to carry out a literature review of a ready-to-use 10% liquid immunoglobulin preparation, Kiovig® (Baxter, Brussels, Belgium), in the treatment of primary immunodeficiency.

Methods

Studies were identified by searching PubMed, Centre for Reviews and Dissemination databases, Cochrane Database of Systematic Reviews, and EconLit up to January 2010. The clinical literature review focused on studies of the safety, tolerability, and effectiveness of Kiovig. Evidence about cost-effectiveness was derived from economic evaluations. In addition, budget impact analyses were identified that examined the financial impact of adopting Kiovig for the treatment of primary immunodeficiency.

Results

The evidence indicates that Kiovig and other intravenous immunoglobulin products have similar safety and effectiveness. Given that Kiovig and other intravenous immunoglobulin products appear to have similar effectiveness, the pharmacoeconomic value of Kiovig depends on the costs of immunoglobulin treatment, including drug acquisition costs, and pharmacist and nursing time costs. A Belgian study indicated that the price of Kiovig is the main driver of its budget impact on the treatment of primary immunodeficiency.

Conclusion

The current evidence base on the treatment of primary immunodeficiency with Kiovig is limited. Head-to-head comparative studies are called for to investigate the safety, tolerability, effectiveness, and cost-effectiveness of Kiovig versus other immunoglobulin products in the treatment of primary immunodeficiency.

     

Pharmaceutical pricing, price controls, and their effects on pharmaceutical sales and research and development expenditures in the European Union

BACKGROUND: Each country in the European Union (EU) currently employs direct price controls or permutations of direct price controls, such as reference pricing or limitations on returns to capital. Some countries also use volume controls. A new proposal that is being discussed would have all of the countries in the EU adopt uniform pricing for each pharmaceutical. OBJECTIVE: This paper analyzes the economic effects of free-market pricing individual-country price controls, and uniform EU price controls. METHODS: Microeconomic and mathematical models were used to simulate and predict probable economic outcomes in a comparative static setting. RESULTS: Price controls may be in the form of price ceilings or price floors. Both forms of price control generate deadweight economic losses in the short run and long run. A uniform EU price for each pharmaceutical sold there would have elements of a price ceiling in some of the countries and of a price floor in other countries. The deadweight loss incurred would be a function of the level at which the uniform price was set by the EU and the price elasticity of demand for each pharmaceutical in each country. CONCLUSIONS: Economic efficiency is maximized in both the short run and long run when prices are set in freely competitive markets. An additional important dimension of Ramsey pricing within a competitive context is that it generates funds for investment in pharmaceutical research and development, which enhances economic efficiency in the long run.     

Values‐based practice and phenomenological psychopathology: implications of existential changes in depression

Values‐based practice (VBP), developed as a partner theory to evidence‐based medicine (EBM), takes into explicit consideration patients' and clinicians' values, preferences, concerns and expectations during the clinical encounter in order to make decisions about proper interventions. VBP takes seriously the importance of life narratives, as well as how such narratives fundamentally shape patients' and clinicians' values. It also helps to explain difficulties in the clinical encounter as conflicts of values. While we believe that VBP adds an important dimension to the clinician's reasoning and decision‐making procedures, we argue that it ignores the degree to which values can shift and change, especially in the case of psychiatric disorders. VBP does this in three respects. First, it does not appropriately engage with the fact that a person's values can change dramatically in light of major life events. Second, it does not acknowledge certain changes in the way people value, or in their modes of valuing, that occur in cases of severe psychiatric disorder. And third, it does not acknowledge the fact that certain disorders can even alter the degree to which one is capable of valuing anything at all. We believe that ignoring such changes limits the degree to which VBP can be effectively applied to clinical treatment and care. We conclude by considering a number of possible remedies to this issue, including the use of proxies and written statements of value generated through interviews and discussions between patient and clinician.     

A Novel Method to Value Real Options in Health Care: The Case of a Multicohort Human Papillomavirus Vaccination Strategy

Background

A large number of economic evaluations have already confirmed the cost-effectiveness of different human papillomavirus (HPV) vaccination strategies. Standard analyses might not capture the full economic value of novel vaccination programs because the cost-effectiveness paradigm fails to take into account the value of active management. Management decisions can be seen as real options, a term used to refer to the application of option pricing theory to the valuation of investments in nonfinancial assets in which much of the value is attributable to flexibility and learning over time.

Objective

The aim of this article was to discuss the potential advantages shown by using the payoff method in the valuation of the cost-effectiveness of competing HPV immunization programs.

Methods

This was the first study, to the best of our knowledge, to use the payoff method to determine the real option values of 4 different HPV vaccination strategies targeting female subjects aged 12, 15, 18, and 25 years. The payoff method derives the real option value from the triangular payoff distribution of the project's net present value, which is treated as a triangular fuzzy number. To inform the real option model, cost-effectiveness data were derived from an empirically calibrated Bayesian model designed to assess the cost-effectiveness of a multicohort HPV vaccination strategy in the context of the current cervical cancer screening program in Italy. A net health benefit approach was used to calculate the expected fuzzy net present value for each of the 4 vaccination strategies evaluated.

Results

Costs per quality-adjusted life-year gained seemed to be related to the number of cohorts targeted: a single cohort of girls aged 12 years (€10,955 [95% CI, –1,021 to 28,212]) revealed the lowest cost among the 4 alternative strategies evaluated. The real option valuation challenged the cost-effectiveness dominance of a single cohort of 12-year-old girls. The simultaneous vaccination of 2 cohorts of girls aged 12 and 15 years yielded a real option value (€17,723) equivalent to that attributed to a single cohort of 12-year-old girls (€17,460).

Conclusions

The payoff method showed distinctive advantages in the valuation of the cost-effectiveness of competing health care interventions, essentially determined by the replacement of the nonfuzzy numbers that are commonly used in cost-effectiveness analysis models, with fuzzy numbers as an input to inform the real option pricing method. The real option approach to value uncertainty makes policy making in health care an evolutionary process and creates a new “space” for decision-making choices.     

实时心肌声学造影定量评价不同程度心肌缺血的实验研究

目的：探讨实时心肌声学造影定量诊断不同程度心肌缺血的可行性。方法：12只开胸犬以冠脉缩窄器造成左前降支轻，中，重不同程度狭窄，狭窄前后静滴“全氟显”（速度5ml/min)进行实时心肌声学造影。左室乳头肌中段短轴观察左室前壁心肌显影，室壁厚度和运动，采用动态图像单帧转化法以图像软件分析造影结果。结果：轻度缺血时，心肌显影的视频亮度峰值（VBP）及再灌注速度与狭窄前比较无显著差异，药物负荷状态下差异增大；中，重度缺血时二者明显减低。心肌缺血时，舒张期与收缩期VBP比值正常减低。结论：结合药物负荷，实时心肌声学造影可以定量诊断不同程度心肌缺血。     

Cost-Effectiveness and Net Monetary Benefit of Olaparib Maintenance Therapy Versus No Maintenance Therapy After First-line Platinum-based Chemotherapy in Newly Diagnosed Advanced BRCA1/2-mutated Ovarian Cancer in the Italian National Health Service

PurposeThe aim of this study was to evaluate the cost-effectiveness and net monetary benefit of olaparib maintenance therapy compared with no maintenance therapy after first-line platinum-based chemotherapy in newly diagnosed advanced BRCA1/2-mutated ovarian cancer from the Italian National Health Service (NHS) perspective.MethodsWe developed a lifetime Markov model in which a cohort of patients with newly diagnosed advanced BRCA1/2-mutated ovarian cancer was assigned to receive either olaparib maintenance therapy or active surveillance (Italian standard of care) after first-line platinum-based chemotherapy to compare cost-effectiveness and net monetary benefit of the 2 strategies. Data on clinical outcomes were obtained from related clinical trial literature and extrapolated using parametric survival analyses. Data on costs were derived from Italian official sources and relevant real-world studies. The incremental cost-effectiveness ratio (ICER), incremental cost-utility ratio (ICUR), and incremental net monetary benefit (INMB) were computed and compared against an incremental cost per quality-adjusted life-year (QALY) gained of €16,372 willingness-to-pay (WTP) threshold. We used deterministic sensitivity analysis (DSA) and probabilistic sensitivity analysis (PSA) to assess how uncertainty affects results; we also performed scenario analyses to compare results under different pricing settings.FindingsIn the base-case scenario, during a 50-year time horizon, the total costs for patients treated with olaparib therapy and active surveillance were €124,359 and €97,043, respectively, and QALYs gained were 7.29 and 4.88, respectively, with an ICER of €9,515 per life-year gained, an ICUR of €11,345 per QALY gained, and an INMB of €12,104. In scenario analyses, considering maximum selling prices for all other drugs, ICUR decreased to €11,311 per QALY and €7,498 per QALY when a 10% and 20% discount, respectively, was applied to the olaparib official price, and the INMB increased to €12,186 and €21,366, respectively. DSA found that the model results were most sensitive to the proportion of patients with relapsing disease in response to platinum-based chemotherapy, time receiving olaparib first-line maintenance treatment, and subsequent treatments price. According to PSAresults, olaparib was associated with a probability of being cost-effective at a €16,372 per QALY WTP threshold ranging from 70% to 100% in the scenarios examined.ImplicationsOur analysis indicates that olaparib maintenance therapy may deliver a significant health benefit with a contained upfront cost during a 50-year time horizon, from the Italian NHS perspective, providing value in a setting with curative intent.     

Cost-effectiveness of screening the average-risk population for colorectal cancer

This article reviews several of the recent models addressing the cost-effectiveness of colorectal cancer screening in the average-risk individual (Table 1). How can clinicians and policy makers use this information for decision making regarding colorectal cancer screening? The cost-effectiveness ratios reported by themselves do not identify cost-effective practices. They must be placed in a decision context that is expressed in one of two forms. In the first form, an explicit threshold or maximum amount that a policy maker is willing to spend is stated (e.g., $40,000 per LY gained, as has been quoted as an acceptable amount for a prevention program). In the second form of decision context, a list of medical practices and their associated cost-effectiveness ratios, also known as a league table (Table 2) is used as a basis for comparison with the practice under evaluation (e.g., colorectal cancer screening). The practice with the lowest cost-effectiveness ratio is the most cost-effective practice on the list. Practices with lower cost-effectiveness ratios are considered cost-effective compared with those with higher ratios. Table 2 lists incremental cost-effectiveness ratios for common medical practices. The models discussed in this article suggested that colorectal cancer screening using annual FOBT, flexible sigmoidoscopy at 3 or 5 years, the combination of FOBT and flexible sigmoidoscopy, barium enema, colonoscopy, and even virtual colonoscopy had incremental cost-effectiveness ratios ranging from $6300 to $92,900 per LY saved with most of the cost-effectiveness ratio ranging from $10,000 to $40,000 per LY saved. These ratios are similar to the cost of another widely accepted practice, breast cancer screening with annual mammography in women age 50 and older ($22,000 per LY gained). Colorectal cancer screening with any of the modalities discussed is considered less cost-effective than screening for hemochromatosis, which has an incremental cost-effectiveness ratio of $3665 per LY saved. Based on these ratios, however, screening for colorectal cancer is considered cost-effective compared with cervical cancer screening in women age 20 and older with pap smear every 3 years, which has an incremental cost-effectiveness ratio of $250,000 per LY gained. The clinician can use these incremental cost-effectiveness ratios to evaluate the risks and benefits of alternative practices for the individual, and the policy maker with a limited health care budget can use these ratios to set priorities for funding based on the costs and the expected gains in life expectancy for colorectal cancer screening and for alternative health care programs.     

A critical narrative analysis of shared decision-making in acute inpatient mental health care

Shared decision-making (SDM) is a high priority in healthcare policy and is complementary to the recovery philosophy in mental health care. This agenda has been operationalised within the Values-Based Practice (VBP) framework, which offers a theoretical and practical model to promote democratic interprofessional approaches to decision-making. However, these are limited by a lack of recognition of the implications of power implicit within the mental health system. This study considers issues of power within the context of decision-making and examines to what extent decisions about patients’ care on acute in-patient wards are perceived to be shared. Focus groups were conducted with 46 mental health professionals, service users, and carers. The data were analysed using the framework of critical narrative analysis (CNA). The findings of the study suggested each group constructed different identity positions, which placed them as inside or outside of the decision-making process. This reflected their view of themselves as best placed to influence a decision on behalf of the service user. In conclusion, the discourse of VBP and SDM needs to take account of how differentials of power and the positioning of speakers affect the context in which decisions take place.     

Health care value-based purchasing

Two Institute of Medicine studies have focused attention on the glaring deficiencies in health care safety and quality. These studies and others serve as wake-up calls within both the private and government-sponsored United States health care industry. As a result of the "quality chasm" and crippling rise in health care cost, the concept of value-based purchasing (VBP) has gained increasing momentum. The stakeholders affected by the health care value equation include consumers, employers, insurers, and providers. VBP creates quality standards and stakeholder incentives to improve quality of care and become more cost efficient. The greatest challenge is the development of methods and information sources that will allow reliable, accurate, and credible measures of health care quality. VBP presents the ambitious hope of creating transparent measures of quality and cost efficiency that would allow the consumer to make health care purchasing decisions. If this can be achieved, then the competitive forces that increase value in other industries will function in health care.     

Impact of formulary restrictions on the cost-effectiveness of antidepressant treatment

Newer antidepressants are associated with higher costs of treatment of anxiety and depression. Managed care organizations are challenged to control treatment costs by implementing restricted formularies based on price and perceived medical value. Despite unfavorable side effects of efficacious tricyclic antidepressants, the low acquisition cost rationalizes the inclusion of this older class of agents on a formulary. On the other hand, cost-containment approaches have been taken toward more expensive drug classes (e.g., selective serotonin reuptake inhibitors) despite a superior safety profile of these drug classes over tricyclics. There is compelling evidence that dual reuptake inhibitors (e.g., venlafaxine extended-release), which have acquisition costs similar to serotonin reuptake inhibitors, have a broad spectrum of efficacy and thus added value, contributing to the cost-effectiveness of including this agent in the managed care formulary. Assessment of overall cost-effectiveness should not be limited by acquisition costs but should take total healthcare costs into consideration.     

G-CSF for the prophylaxis of neutropenic fever in patients with small cell lung cancer receiving myelosuppressive antineoplastic chemotherapy: meta-analysis and pharmacoeconomic evaluation

Standard meta-analytical and pharmacoeconomic techniques were used to study the clinical effectiveness and the cost-effectiveness ratio of the prophylactic (or pre-emptive) administration of G-CSF to patients with small cell lung cancer treated with conventional myelosuppressive cyto-toxic chemotherapy. In the first part of our study, we conducted a meta-analysis of the randomized clinical trials evaluating G-CSF for this clinical indication. Three trials were identified by our literature search and were included in the meta-analysis (overall number of patients = 606). The end-points for evaluating G-CSF included mortality from infection and the cumulative incidence of neutropenic fever over six cycles of chemotherapy. The results of our meta-analysis demonstrate that prophylactic G-CSF did not affect mortality but significantly reduced the incidence of neutropenic fever from 68·3% to 38·7% (pooled odds ratio = 0·29, 95% CI: 0·21-0·40; P <0·001). In the second part of our study, we carried out a pharmacoeconomic analysis to estimate the cost-effectiveness ratio of pre-emptive G-CSF (i. e. the 'average' cost associated with the prevention of an episode of neutropenic fever). This cost-effectiveness ratio was US$ 14 372 using the Italian price of the drug converted into dollars, or US$ 41 088 using the US price. Finally, we estimated the revenue-neutral price of G-CSF based on American data of the cost-of-illness. This price ranged from US$ 395 to US$ 569 per cycle, a figure higher than the value (US$ 150) previously reported in the literature.     

Health technology assessment and its role in the future development of the Indian healthcare sector

Public expenditure on healthcare in India is low by international comparison, and access to essential treatment pushes many uninsured citizens below the poverty line. In many countries, policymakers utilize health technology assessment (HTA) methodologies to direct investments in healthcare, to obtain the maximum benefit for the population as a whole. With rising incomes and a commitment from the Government of India to increase the proportion of gross domestic product spent on health, this is an opportune moment to consider how HTA might help to allocate healthcare spending in India, in an equitable and efficient manner. Despite the predominance of out-of-pocket payments in the Indian healthcare sector, payers of all types are increasingly demanding value for money from expenditure on healthcare. In this review we demonstrate how HTA can be used to inform several aspects of healthcare provision. Areas in which HTA could be applied in the Indian context include, drug pricing, development of clinical practice guidelines, and prioritizing interventions that represent the greatest value within a limited budget. To illustrate the potential benefits of using the HTA approach, we present an example from a mature HTA market (Canada) that demonstrates how a new treatment for patients with atrial fibrillation - although more expensive than the current standard of care - improves clinical outcomes and represents a cost-effective use of public health resources. If aligned with the prevailing cultural and ethical considerations, and with the necessary investment in expert staff and resources, HTA promises to be a valuable tool for development of the Indian healthcare sector.     

Economic evaluation of a nursing-led inpatient unit: the impact of findings on management decisions of service utility and sustainability

AIMS: The nursing-led inpatient unit is designed to substitute for a period of care in acute hospital wards and to improve patient outcome prior to discharge to the community. This paper aims to evaluate the cost, from the UK National Health Service perspective, of transfer to a nursing-led inpatient unit for intermediate care and to discuss the impact of these findings to the future development and sustainability of the nursing-led inpatient unit. BACKGROUND: Recent economic analyses have showed that nursing-led inpatient units are associated with increased costs of care with length of stay as the main driver of inpatient costs. METHOD: The cost-effectiveness analysis was part of a randomized-controlled trial with a sample size of 175, of which 89 were in the nursing-led inpatient unit arm and 86 in the control arm. Resource use data included length of stay, investigations performed, multiprofessional input and nursing input. Clinical outcome was measured using Barthel Index, a functional status measure. RESULTS: Cost per day was lower on the nursing-led inpatient unit although cost per hospital stay was higher due to significantly increased length of stay. Postdischarge community care costs were lower. The incremental cost-effectiveness ratio of the treatment was 1044 pounds sterling per point improvement of the Barthel Index. CONCLUSIONS: The nursing-led inpatient unit was associated with higher costs however, the question of whether the nursing-led inpatient unit is cost-effective has not been clearly answered because of the limited follow-up period of the study. The increased cost of care on the nursing-led inpatient unit was not a major factor in local management decisions about the future of the unit. The changes in the context of service provision within which the nursing-led inpatient unit operated as a result of substantial investment in intermediate care did have a major impact.     

Three-Year Cost-effectiveness Model for Non–Animal Stabilized Hyaluronic Acid and Dextranomer Copolymer Compared With Sacral Nerve Stimulation After Conservative Therapy for the Management of Fecal Incontinence

Background

Two new therapies for fecal incontinence (FI) are now available: non–animal stabilized hyaluronic acid and dextranomer copolymer (NASHA/Dx) and sacral nerve stimulation (SNS).

Purpose

This study aimed to determine the cost-effectiveness of NASHA/Dx compared with SNS and conservative therapy (CT) for the treatment of FI after CT failure.

Methods

Decision tree models with Markov subbranches were developed to compare all direct costs and outcomes during a 3-year period from the viewpoint of the US third-party payer. Costs (in 2013 US dollars) of devices, medical and surgical care, and hospitalization were included. Outcomes included quality-adjusted life-years (QALYs) and incontinence-free days (IFDs). Both costs and outcomes were discounted at an annual rate of 3%. The incremental cost-effectiveness ratio was calculated for each outcome. One-way and probabilistic sensitivity analyses were performed to examine robustness of results and model stability. A budget impact analysis was also undertaken to estimate the potential cost and savings of NASHA/Dx for a payer with 1,000,000 covered lives.

Results

For the 3-year cost-effectiveness models, the expected cost was $9053 for CT, $14,962 for NASHA/Dx, and $33,201 for SNS. The numbers of QALYs were 1.769, 1.929, and 2.004, respectively. The numbers of IFDs were 128.8, 267.6, and 514.8, respectively. The incremental cost-effectiveness ratios per additional IFD gained were $42.60 for NASHA/Dx vs CT, $73.76 for SNS vs NASHA/Dx, and $62.55 for SNS vs CT. The incremental costs per QALY gained were $37,036 for NASHA/Dx vs CT, $244,509 for SNS vs NASHA/Dx, and $103,066 for SNS vs CT. The budget impact analysis evaluated the financial effect on the health care system of the use of NASHA/Dx and SNS. For the scenarios evaluated, when all of the patients receive NASHA/Dx, the net annual effect to the health care payer budget ranged from $571,455 to $2,857,275. When all of the patients receive SNS, the net annual effect to the health care payer budget ranged from $1,959,323 to $9,796,613.

Conclusion

Both NASHA/Dx and SNS have produced significant improvements in FI symptoms for affected patients. NASHA/Dx is a cost-effective and more efficient use of resources for the treatment of FI when compared with SNS. The budget impact analysis suggests that although reimbursement for NASHA/Dx treatment initially adds costs to the health care system, it is significantly less expensive than SNS for patients who are candidates for either treatment.     

Cost-Effectiveness of Everolimus for Second-Line Treatment of Metastatic Renal Cell Carcinoma in Serbia

Background

New targeted therapeutics for metastatic renal cell carcinoma (mRCC) enable an increment in progression-free survival (PFS) ranging from 2 to 6 months. Compared with best supportive care, everolimus demonstrated an additional PFS of 3 months in patients with mRCC whose disease had progressed on sunitinib and/or sorafenib. The only targeted therapy for mRCC currently reimbursed in Serbia is sunitinib.

Objective

The aim of this study was to estimate the cost-effectiveness and the budget impact of the introduction of everolimus in Serbia in comparison to best supportive care, for mRCC patients refractory to sunitinib.

Methods

A Markov model was designed corresponding with Serbian treatment protocols. A health care payer perspective was taken, including direct costs only. Treated and untreated cohorts were followed up over 18 cycles, each cycle lasting 8 weeks, which covered the lifetime horizon of mRCC patients refractory to the first-line treatment. Annual discounted rates of 1.5% for effectiveness and 3% for costs were applied. Transitions between health states were modeled by time-dependent probabilities extracted from published Kaplan-Meier curves of PFS and overall survival (OS). Utility values were obtained from the appraisals of other mRCC treatments. One-way and probabilistic sensitivity analyses were done to test the robustness and uncertainty of the base–case estimate. Lastly, the potential impacts of everolimus on the overall health care expenditures on annual and 4-year bases were estimated in the budget-impact analysis.

Results

The incremental cost-effectiveness ratio for everolimus was estimated at €86,978 per quality-adjusted life-year. Sensitivity analysis identified the hazard multiplier, a statistical approximator of OS gain, as the main driver of everolimus cost-effectiveness. Furthermore, probabilistic sensitivity analyses revealed a wide 95% CI around the base–case incremental cost-effectiveness ratio estimate (€32,594–€425,258 per quality-adjusted life-year). Finally, an average annual budgetary impact of everolimus in first 4 years after its potential reimbursement would be around €270,000, contributing to <1% of the total budget in Serbian oncology.

Conclusions

Everolimus as a second-line treatment of mRCC is not likely to be a cost-effective option under the present conditions in Serbia, with a relatively limited impact on its budget in oncology. A major constraint on the estimation of the cost-effectiveness of everolimus relates to the uncertainty around the everolimus effect on extending OS. However, prior to a final decision on the acceptance/rejection of everolimus, reassessment of the whole therapeutic group might be needed to construct an economically rational treatment strategy within the mRCC field.     

A COST-EFFECTIVENESS MODEL OF ALTERNATIVE STATINS TO ACHIEVE TARGET LDL-CHOLESTEROL LEVELS

An economic model was developed to estimate the relative cost-effectiveness of alternative HMG-CoA reductase inhibitors (statins) – atorvastatin, cerivastatin, fluvastatin, pravastatin and simvastatin – to achieve target low-density lipoprotein cholesterol (LDL-C) levels in a population of secondary CHD prevention patients. By using a cholesterol target as the endpoint of interest and a dose titration approach, the model assumes that the statins demonstrate a class effect through cholesterol lowering. The model was used to estimate the proportion of patients achieving target LDL-C levels (<3 mmol/l) under each scenario tested. Total costs and incremental cost-effectiveness relative to no treatment and to the lowest cost option were estimated for each scenario. Total costs were highest for pravastatin and lowest for cerivastatin. Compared with no treatment, the incremental cost per patient treated to target LDL-C varied between £383 (atorvastatin) and £1213 (pravastatin). Incremental cost-effectiveness ratios in comparison with the lowest cost treatment (cerivastatin) were £141 per additional patient achieving target LDL-C with atorvastatin, and £275 with simvastatin. Fluvastatin and pravastatin were both less effective and more expensive than the lowest cost therapy. Although cerivastatin was associated with lowest expected costs, therapy with atorvastatin achieved the lowest cost-effectiveness ratios. Hence atorvastatin would allow the largest number of patients to be treated to target LDL-C within a fixed drug budget. Choosing between drug therapies on the basis of price alone may be misleading if the effectiveness of therapies varies.     

Economic Evaluation of Adding Daratumumab to a Regimen of Bortezomib + Dexamethasone in Relapsed or Refractory Multiple Myeloma: Based on the Latest Updated Analysis of CASTOR

PurposeAdding daratumumab to a regimen of bortezomib + dexamethasone (Vd) has been reported to provide a benefit of longer progression-free survival in patients with relapsed or refractory multiple myeloma (RRMM). However, it is still unclear whether the addition of daratumumab is cost-effective in RRMM. Based on the latest updated analysis of data from the CASTOR trial, this study performed an economic evaluation of the addition of daratumumab to Vd in patients with RRMM.MethodsA Markov decision model was used for estimating the long-term costs and efficacy of Vd with or without daratumumab in patients with RRMM. Data on efficacy were taken from the CASTOR trial to compare Vd + daratumumab with Vd. Costs were taken from the US Centers for Medicare & Medicaid Services and from the literature. A series of sensitivity analyses were performed to address the robustness of the model. Variations in the price of daratumumab and subgroup analyses were conducted.FindingsThe base-case analysis showed that adding daratumumab to Vd provided an additional 1.256 quality-adjusted life-years (QALYs) or 1.645 life-years (LYs), with incremental 213,164 USD (163,184 USD) per QALY (LY) gained. Univariate sensitivity analyses suggested that the subsequent treatment cost of DVd and the price of daratumumab had the greatest effect on the incremental cost-effectiveness ratio. According to the variations analysis of the price of daratumumab, the addition of daratumumab would be cost-effective when daratumumab was priced at 70% (30%) of the current price at a willingness-to-pay threshold of 200,000 USD/QALY (150,000 USD/QALY). Subgroup analysis indicated that adding daratumumab to Vd was most cost-effective in patients with 1 prior line of therapy.ImplicationsFrom a US-payer perspective, daratumumab added to Vd in RRMM is likely to exceed the common accepted values of cost-effectiveness