Mifamurtide for High-Grade,Resectable, Nonmetastatic Osteosarcoma Following Surgical Resection: A Cost-Effectiveness Analysis

ObjectivesMifamurtide is an immune macrophage stimulant that when added to standard chemotherapy has demonstrated survival benefit for newly diagnosed osteosarcoma. The objectives of this study were to investigate the cost-effectiveness of adding mifamurtide to standard three- or four-agent chemotherapy for high-grade, resectable, nonmetastatic osteosarcoma following surgical resection and the issues of obtaining robust cost-effectiveness estimates for ultra-orphan drugs, given the shortage of data.MethodsAn economic evaluation was conducted from the perspective of the UK’s National Health Service as part of the manufacturer’s submission to the National Institute for Health and Care Excellence. The disease process was simplified to a transition through a series of health states, modeled by using a Markov approach. Data to inform the model were derived from patient-level data of Study INT-0133, published literature, and expert opinion. The final efficacy measure was life-years gained (LYG), and utilities were used to obtain quality-adjusted life-years (QALYs).ResultsFor a 60-year time frame and a discount rate of 3.5% for outcomes, patients receiving mifamurtide benefited from an average additional 1.57 years of life and 1.34 QALYs, compared with patients receiving chemotherapy alone, giving an incremental cost-effectiveness ratio (ICER) of £58,737 per LYG and £68,734 per QALY. Because treatment effects were both substantial in restoring health and sustained over a very long period, the National Institute for Health and Care Excellence changed its guidance to allow a discount of 1.5% for outcomes to be applied in these special circumstances. By using this discount factor, it was found that patients receiving mifamurtide had an average additional 2.58 years of life and 2.20 QALYs compared with patients receiving chemotherapy alone, resulting in an ICER of £35,765 per LYG and £41,933 per QALY.ConclusionMifamurtide’s ICER is cost-effective compared with that of other orphan and ultra-orphan drugs, for which prices and corresponding cost-effectiveness estimates are high.     

Cost-Utility Analysis Comparing Heavy-Weight and Light-Weight Mesh in Laparoscopic Surgery for Unilateral Inguinal Hernias

Background

Hernioplasty is one of the most frequent surgeries in the UK. Light-weight mesh (LWM) has the potential to reduce chronic groin pain but its cost-effectiveness compared with heavy-weight mesh (HWM) is unknown.

Objective

Our objective was to conduct a cost-utility analysis between laparoscopic hernioplasty with HWM and LWM for unilateral inguinal hernias.

Methods

A Markov model simulated costs and health outcomes over a period of 1 year (2012) from the societal and National Health Service (NHS) perspective (England). The main outcome was cost per quality-adjusted life-year (QALY) gained. Surgery results were gleaned from the randomized control trial by Bittner et al. Other input parameters were drawn from the literature and public sources of the NHS.

Results

From the societal perspective, LWM induces lower incremental costs (?£88.85) than HWM but yields a slightly smaller incremental effect (?0.00094 QALYs). The deterministic incremental cost-effectiveness ratio (ICER) for HWM compared with LWM amounts to £94,899 per QALY, while the probabilistic ICER is £118,750 (95 % confidence interval [CI] £57,603–180,920). Owing to the withdrawal of productivity losses from the NHS perspective, LWM causes higher incremental costs (£13.09) and an inferior incremental effect (?0.00093), resulting in a dominance of HWM over LWM (ICER 95 % CI ?£12,382 to ?£21,590).

Conclusions

There is no support for the adoption of LWM as standard treatment from an NHS perspective. However, given the small differences between HWM and LWM, LWM has at least the potential of improving patient outcomes and reducing expenditure from the societal perspective.     

A Cost-Effectiveness Framework for Amyotrophic Lateral Sclerosis,Applied to Riluzole

ObjectivesReexamine cost-effectiveness of riluzole in the treatment of amyotrophic lateral sclerosis (ALS) in light of recent advances in disease staging and understanding of stage-specific drug effect.MethodsALS was staged according to the “fine’til 9” (FT9) staging method. Stage-specific health utilities (EQ-5D, US valuation) were estimated from an institutional cohort, whereas literature informed costs and transition probabilities. Costs at 2018 prices were disaggregated into recurring costs (RCs) and “one-off” transition/“tollgate” costs (TCs). Five- and 10-year horizons starting in stage 1 disease were examined from healthcare sector and societal perspectives using Markov models to evaluate riluzole use, at a threshold of $100 000/quality-adjusted life year (QALY). Probabilistic and deterministic sensitivity analyses were conducted.ResultsMean EQ-5D utilities for stages 0 to 4 were 0.79, 0.74, 0.63, 0.54, and 0.46, respectively. From the healthcare sector perspective at the 5-year horizon, riluzole use contributed to 0.182 QALY gained at the cost difference of $12 348 ($5403 riluzole cost, $8870 RC and −$1925 TC differences), translating to an incremental cost-effectiveness ratio (ICER) of $67 658/QALY. Transition probability variation contributed considerably to ICER uncertainty (−30.2% to +90.0%). ICER was sensitive to drug price and RCs, whereas higher TCs modestly reduced ICER due to delayed tollgates.ConclusionThis study provides a framework for health economic studies of ALS treatments using FT9 staging. Prospective stage-specific and disaggregated cost measurement is warranted for accurate future cost-effectiveness analyses. Appropriate separation of TCs from RCs substantially mitigates the high burden of background cost of care on the ICER.     

Cost-Effectiveness of First-Line Pembrolizumab Monotherapy Versus Chemotherapy in High Programmed Death-Ligand 1 Advanced Non–Small Cell Lung Cancer in the Irish Healthcare Setting

ObjectivesThis study aimed to assess the cost-effectiveness of pembrolizumab monotherapy in the first-line treatment of advanced non–small cell lung cancer (NSCLC) in adults whose tumors expressed programmed death-ligand 1 (PD-L1) with a tumor proportion score (TPS) ≥ 50% in the Irish healthcare setting.MethodsEffectiveness inputs were derived from the 5-year analysis of KEYNOTE-024 phase III clinical trial. The intervention was pembrolizumab monotherapy; the comparator was a weighted average of the 5 chemotherapy regimens from the trial. The population included those with previously untreated advanced PD-L1 TPS ≥ 50% NSCLC. A de novo partitioned survival model was developed. Survival modeling was done using Bayesian model averaging on fitted parametric functions. Costs included drug acquisition, treatment initiation, administration and monitoring, adverse events, subsequent treatments, and terminal care. Costs and health state utilities were sourced from the literature and Irish sources. The model had a 20-year time horizon. The perspective taken was the Health Service Executive. A 4% discount rate was applied. Outcomes were expressed as an incremental cost-effectiveness ratio (ICER), measured in terms of incremental costs per quality-adjusted life-year (QALY). Probabilistic sensitivity analysis and 1-way sensitivity analyses were conducted.ResultsThe model estimated a base case ICER of €54 237 per QALY. The probabilistic sensitivity analysis estimated an average ICER of €54 568 per QALY and a 11% probability of cost-effectiveness at the Irish cost-effectiveness threshold of €45 000 per QALY.ConclusionAt the current list price, first-line pembrolizumab monotherapy is not considered cost-effective for the treatment of advanced PD-L1 TPS ≥ 50% NSCLC in the Irish healthcare setting.     

The Economic Value of Innovative Treatments over the Product Life Cycle: The Case of Targeted Trastuzumab Therapy for Breast Cancer

ObjectivePharmacoeconomic analyses typically project the expected cost-effectiveness of a new product for a specific indication. This analysis develops a dynamic life-cycle model to conduct a multiindication evaluation using the case of trastuzumab licensed in the United States for both early-stage and metastatic (or late-stage) human epidermal growth factor receptor 2 (HER2)-positive breast cancer therapy (early breast cancer [EBC]; metastatic breast cancer [MBC]), approved in 2006 and 1998, respectively.MethodsThis dynamic model combined information on expected incremental cost-utility ratios for specific indications with an epidemiologically based projection of utilization by indication over the product life cycle—from 1998 to 2016. Net economic value was estimated as the cumulative quality-adjusted life years (QALYs) gained over the life cycle multiplied by a societal valuation of health gains ($/QALY) minus cumulative net direct treatment costs. Sensitivity analyses were performed under a range of assumptions.ResultsWe projected that the annual number of EBC patients receiving trastuzumab will be more than three times that of MBC by 2016, in part because adjuvant treatment reduces the future incidence of MBC. Over this life cycle, the estimated overall incremental cost-effectiveness ratio (ICER) was $35,590/QALY with a total of 432,547 discounted QALYs gained. Under sensitivity analyses, the overall ICER varied from $21,000 to $53,000/QALY, and the projected net economic value resulting from trastuzumab treatment ranged from $6.2 billion to $49.5 billion.ConclusionsAverage ICERs for multiindication compounds can increase or decrease over the product life cycle. In this example, the projected overall life-cycle ICER for trastuzumab was less than one half of that in the initial indication. This dynamic perspective—versus the usual static one—highlights the interdependence of drug development decisions and investment incentives, raising important reimbursement policy issues.     

Economic Evaluation of Cognitive Behavioral Therapy for Depression: A Systematic Review

ObjectivesThis study aimed to conduct a systematic review of cost-utility studies of internet-based and face-to-face cognitive behavioral therapy (CBT) for depression from childhood to adulthood and to examine their reporting and methodological quality.MethodsA structured search for cost-utility studies concerning CBT for depression was performed in 7 comprehensive databases from their inception to July 2020. Two reviewers independently screened the literature, abstracted data, and assessed quality using the Consolidated Health Economic Evaluation Reporting Standards and Quality of Health Economic Studies checklists. The primary outcome was the incremental cost-effectiveness ratio (ICER) across all studies. To make a relevant comparison of the ICERs across the identified studies, cost data were inflated to the year 2020 and converted into US dollars.ResultsThirty-eight studies were included in this review, of which 26 studies (68%) were deemed of high methodological quality and 12 studies (32%) of fair quality. Despite differences in study designs and settings, the conclusions of most included studies for adult depression were general agreement; they showed that face-to-face CBT monotherapy or combination therapy compared with antidepressants and usual care for adult depression were cost-effective from the societal, health system, or payer perspective (ICER ?$241 212.4/quality-adjusted life-year [QALY] to $33 032.47/QALY, time horizon 12-60 months). Internet-based CBT regardless of guided or unguided also has a significant cost-effectiveness advantage (ICER ?$37 717.52/QALY to $73 841.34/QALY, time horizon 3-36 months). In addition, CBT was cost-effective in preventing depression (ICER ?$23 932.07/QALY to $26 092.02/QALY, time horizon 9-60 months). Nevertheless, the evidence for the cost-effectiveness of CBT for children and adolescents was still ambiguous.ConclusionsFair or high-quality evidence showed that CBT monotherapy or combination therapy for adult depression was cost-effective; whether CBT-related therapy was cost-effective for children and adolescents depression remains inconclusive.     

Cost-Effectiveness of Routine Childhood Vaccination Against Seasonal Influenza in Germany

ObjectivesIn Germany, routine influenza vaccination with quadrivalent influenza vaccines (QIV) is recommended and reimbursed for individuals ≥60 years of age and individuals with underlying chronic conditions. The present study examines the cost-effectiveness of a possible extension of the recommendation to include strategies of childhood vaccination against seasonal influenza using QIV.MethodsA dynamic transmission model was used to examine the epidemiological impact of different childhood vaccination strategies. The outputs were used in a health economic decision tree to calculate the costs per quality-adjusted life year (QALY) gained from a societal and a third-party payer (TPP) perspective. Strain-specific epidemiology, vaccine uptake, and vaccine efficacy data from the 10 non-pandemic seasons from 2003/2004 to 2013/2014 were used, and cost data were drawn mainly from a health insurance claims data analysis and supplemented by estimates from literature. Uncertainty is explored via scenario, deterministic, and probabilistic sensitivity analyses.ResultsVaccinating 2- to 9-year-olds with QIV assuming a vaccine uptake of 40% is cost-saving with a benefit–cost ratio of 1.66 from a societal perspective and an incremental cost-effectiveness ratio of €998/QALY from a TPP perspective. Lower and higher vaccine uptakes show marginal effects, while extending the target group to 2- to 17-year-olds further increases the health benefits while still being below the willingness-to-pay (WTP) threshold. Assuming no vaccine-induced herd protection has a negative effect on the cost-effectiveness ratio, but childhood vaccination remains cost-effective.ConclusionRoutine childhood vaccination against seasonal influenza in Germany is most likely to be cost-saving from a societal perspective and highly cost-effective from a TPP perspective.     

Cost-Effectiveness of Venetoclax Plus Obinutuzumab Versus Chlorambucil Plus Obinutuzumab for the First-Line Treatment of Adult Patients With Chronic Lymphocytic Leukemia: An Extended Societal View

ObjectivesEfficacy of venetoclax plus obinutuzumab (VenO) compared with chlorambucil plus obinutuzumab (ClbO) for treatment-naïve adult patients with chronic lymphocytic leukemia (CLL) with coexisting medical conditions was investigated in CLL14 (NCT02242942). Our aim was to evaluate the cost-effectiveness of VenO versus ClbO for these patients from a Dutch societal perspective.MethodsA 3-state partitioned survival model was constructed to evaluate the cost-effectiveness of VenO. The outcome of the analysis was the incremental cost-effectiveness ratio (ICER) with effectiveness measured in quality-adjusted life-years (QALYs) gained. Uncertainty was explored through deterministic and probabilistic sensitivity analyses, scenario analyses, and value of information analysis (VOI).ResultsThe base case resulted in a discounted ICER −49 928 EUR/QALY gained (with incremental negative costs and positive effects). None of the ICERs resulted from deterministic sensitivity and scenario analyses exceeded the chosen willingness-to-pay threshold of 20 000 EUR/QALY, and > 99% of the iterations in the probabilistic sensitivity analysis were cost-effective. VOI analyses showed a maximum expected value of eliminating all model parameter uncertainty of 183 591 EUR.ConclusionsOur study demonstrated VenO being dominant over ClbO in treatment-naïve adult patients with CLL assuming a Dutch societal perspective. We concluded that our results are robust as tested through sensitivity and scenario analyses. Additionally, the VOI analyses confirmed that our current evidence base is strong enough to generate reliable results for our study. Nevertheless, further research based on real-world data or longer follow-up period could further contribute to the robustness of the current study’s conclusions.     

Stratified cost-utility analysis of total hip arthroplasty in displaced femoral neck fracture

ObjectiveTo conduct a stratified cost-utility analysis of total versus partial hip arthroplasty as a function of clinical subtype.MethodAll cases of this type of intervention were analysed between 2010 and 2016 in the Basque Health Service, gathering data on clinical outcomes and resource use to calculate the cost and utility in quality-adjusted life years (QALYs) at individual level. The statistical analysis included applying the propensity score to balance the groups, and seemingly unrelated regression models to calculate the incremental cost-utility ratio and plot the cost-effectiveness plane. The interaction between age group and American Society of Anesthesiologists (ASA) risk class was assessed in the multivariate analysis.ResultsThe study identified 5867 patients diagnosed with femoral neck fracture, of whom 1307 and 4560 were treated with total and partial hip arthroplasty, respectively. In the cost-utility analysis based on the seemingly unrelated regression, total hip arthroplasty was found to have a higher cost and higher utility (2465€ and 0.42 QALYs). Considering a willingness-to-pay threshold of €22,000 per QALY, total hip arthroplasty was cost-effective in the under-80-year-old subgroup. Among patients above this age, hemiarthroplasty was cost-effective in ASA class I-II patients and dominant in ASA class III-IV patients.ConclusionsSubgroup analysis supports current daily clinical practice in displaced femoral neck fractures, namely, using partial replacement in most patients and reserving total replacement for younger patients.     

Cost-effectiveness of adding oseltamivir to primary care for influenza-like-illness: economic evaluation alongside the randomised controlled ALIC4E trial in 15 European countries

Background

Oseltamivir is usually not often prescribed (or reimbursed) for non-high-risk patients consulting for influenza-like-illness (ILI) in primary care in Europe. We aimed to evaluate the cost-effectiveness of adding oseltamivir to usual primary care in adults/adolescents (13 years +) and children with ILI during seasonal influenza epidemics, using data collected in an open-label, multi-season, randomised controlled trial of oseltamivir in 15 European countries.

Methods

Direct and indirect cost estimates were based on patient reported resource use and official country-specific unit costs. Health-Related Quality of Life was assessed by EQ-5D questionnaires. Costs and quality adjusted life-years (QALY) were bootstrapped (N = 10,000) to estimate incremental cost-effectiveness ratios (ICER), from both the healthcare payers’ and the societal perspectives, with uncertainty expressed through probabilistic sensitivity analysis and expected value for perfect information (EVPI) analysis. Additionally, scenario (self-reported spending), comorbidities subgroup and country-specific analyses were performed.

Results

The healthcare payers’ expected ICERs of oseltamivir were €22,459 per QALY gained in adults/adolescents and €13,001 in children. From the societal perspective, oseltamivir was cost-saving in adults/adolescents, but the ICER is €8,344 in children. Large uncertainties were observed in subgroups with comorbidities, especially for children. The expected ICERs and extent of decision uncertainty varied between countries (EVPI ranged €1–€35 per patient).

Conclusion

Adding oseltamivir to primary usual care in Europe is likely to be cost-effective for treating adults/adolescents and children with ILI from the healthcare payers’ perspective (if willingness-to-pay per QALY gained > €22,459) and cost-saving in adults/adolescents from a societal perspective.

     

Cost-effectiveness of vaccination of the elderly against herpes zoster in The Netherlands

Background

Each year a substantial number of Dutch elderly suffers from herpes zoster (HZ), caused by the reactivation of the varicella zoster virus (VZV). A potential complication of HZ is postherpetic neuralgia (PHN) which results in a prolonged loss of quality of life. A large randomized clinical trial, labelled the Shingles Prevention study (SPS), demonstrated that a live attenuated VZV vaccine can reduce the incidence of HZ and PHN.

Objective

We aimed to estimate the incremental cost-effectiveness ratio (ICER) of vaccination of the elderly against HZ versus no such vaccination in The Netherlands.

Methods

A cohort model was developed to compare the costs and effects in a vaccinated and a non-vaccinated age- and gender-stratified cohort of immune-competent elderly. Vaccination age was varied from 60 to 75 years. Data from published literature such as the SPS were used for transition probabilities. The study was performed from the societal as well as the health care payer's perspective and results were expressed in euros per quality-adjusted life year (QALY) gained.

Results

In the base case, we estimated that vaccination of a cohort of 100,000 60-year-olds would prevent 4136 cases of HZ, 305 cases of PHN resulting in a QALY-gain of 209. From the societal perspective, a total of €1.9 million was saved and the ICER was €35,555 per QALY gained when a vaccine price of €87 was used. Vaccination of women resulted in a lower ICER than vaccination of men (€33,258 vs. €40,984 per QALY gained). The vaccination age with the most favourable ICER was 70 years (€29,664 per QALY gained). Parameters with a major impact on the ICER were the vaccine price and HZ incidence rates. In addition, the model was sensitive to utility of mild pain, vaccine efficacy at the moment of uptake and the duration of protection induced by the vaccine.

Conclusion

Vaccination against HZ might be cost-effective for ages ranging from 60 to 75 when a threshold of €50,000 per QALY gained would be used, at €20,000 per QALY this might not be the case. Additional information on the duration of vaccine-protection is needed to further optimize cost-effectiveness estimations.     

Cost-effectiveness analysis of influenza vaccination during pregnancy in Japan

BackgroundPregnant women and infants are known as high risk groups for influenza. WHO recommend pregnant women be vaccinated with inactivated influenza vaccine. In Japan, some municipalities started to give subsidy to encourage pregnant women to receive a shot on their own accord, which has made the introduction of seasonal antepartum maternal vaccination program (AMVP) into the routine vaccination list a current topic in health policy and has raised the need to evaluate the value for money of such possibility.MethodsWe conducted a cost-effectiveness analysis to evaluate the efficiency of conducting AMVP in Japan. A decision tree model was adopted taking into consideration the duration of single-year vaccine effectiveness for infants and for mothers. The program targeted pregnant women aged 20–49 years old at or over 12 weeks gestation during October 1 through March 30. Estimated probabilities of treatments received due to influenza for pregnant/postpartum women or their infants varied by calendar time, vaccination status, and/or gestational age. Incremental cost-effectiveness ratio (ICER) compared with current no-AMVP from societal perspective was calculated. Transition probabilities, utility weights to estimate quality-adjusted life year (QALY), and disease treatment costs were either calculated or extracted from literature. Costs per vaccination was assumed at ¥3,529/US$32.1.ResultsAMVP reduces disease treatment costs, while the reduction cannot offset the vaccination cost. Incremental QALYs were at 0.00009, among them 84.2% were from infants. ICER was ¥7,779,356/US$70,721 per QALY gained. One-way sensitivity analyses revealed that vaccine effectiveness for infant and costs per shot were the two main key variables affecting the ICER.ConclusionWe found that vaccinating pregnant women with influenza vaccine to prevent unvaccinated infants and pregnant/postpartum women from influenza-associated disease in Japan can be cost-effective from societal perspective, under the WHO-suggested “cost-effective” criteria (1–3 times of GDP).     

Cost effectiveness of typhoid vaccination in India

IntroductionWorld Health Organization has prequalified the use of typhoid conjugate vaccine (TCV) in children over six months of age in typhoid endemic countries. We assessed the cost-effectiveness of introducing TCV separately for urban and rural areas of India.MethodsA decision analytic model was developed, using a societal perspective, to compare long-term costs and outcomes (3% discount rate) in a new-born cohort of 100,000 children immunized with or without TCV. Three vaccination scenarios were modelled, assuming the protective efficacy of TCV to last for 5, 10 and 15 years following immunization. Incidence of typhoid infection estimated under ‘National Surveillance System for Enteric Fever’ (NSSEFI)’ was used. The prices of vaccine and cost of service delivery were included for vaccination arm. Both health system cost and out-of-pocket expenditures for treatment of typhoid illness and its complications was included.ResultsTCV introduction in urban areas would result in prevention of 17% to 36% typhoid cases and deaths. With exclusion of indirect costs, the incremental cost per QALY gained was ₹ 151,346 (54,730–307,975), ₹ 61,710 (−5250 to 163,283) and ₹ 45,188 (−17,069 to 141,093) for scenario 1, 2 and 3 respectively. While, with inclusion of indirect costs, all 3 scenarios were cost saving. Further, in rural areas, TCV is estimated to reduce the typhoid cases and deaths by 19% to 36%, with ICER (incremental cost per QALY gained) ranging from ₹ 2340 (1316–4370) to ₹ 3574 (2057 – 6691) thousand (inclusive of indirect costs) among the 3 vaccination scenarios.ConclusionFrom a societal perspective, introduction of TCV is a cost saving strategy in urban India. Further, due to low incidence of typhoid infection, introduction of TCV is not cost-effective in rural settings of India.     

Cost-utility analysis from a societal perspective

In this paper, we outline how to use cost-utility analysis from a societal perspective and the arguments that could be made for using data such as a model for economic evaluation of health care. We show that to include all the costs in the analysis, a price per quality-adjusted life years (QALY) gained rather than a given budget should be used as the decision rule. Using cost-utility analysis this way is based on a willingness to pay per QALY gained that is constant and the same for everyone. To use a fixed price per QALY gained is consistent with societal utility maximization if aggregated QALYs are a measure of societal utility and if the mix of financing sources is the same for all health care programmes. If, furthermore, the price per QALY gained is set at the optimal level, cost-utility analysis will lead to a maximization of societal utility. To get more information on the willingness to pay per QALY gained so as to provide cost-utility analysis with a useful decision rule should be a research priority.     

Economic Evaluation of Cinacalcet in the United States: The EVOLVE Trial

BackgroundPrevious economic evaluations of cinacalcet in patients with secondary hyperparathyroidism (sHPT) relied on the combination of surrogate end points in clinical trials and epidemiologic studies.ObjectivesThe objective was to conduct an economic evaluation of cinacalcet on the basis of the EValuation Of Cinacalcet HCl Therapy to Lower CardioVascular Events (EVOLVE) trial from a US payer perspective.MethodsWe developed a semi-Markov model to assess the cost-effectiveness of cinacalcet in addition to conventional therapy, compared with conventional therapy alone, in patients with moderate-to-severe sHPT receiving hemodialysis. We used treatment effect estimates from the unadjusted intent-to-treat (ITT) analysis and prespecified covariate-adjusted ITT analysis as our main analyses. We assessed model sensitivity to variations in individual inputs and overall decision uncertainty through probabilistic sensitivity analyses.ResultsThe incremental cost-effectiveness ratio (ICER) for cinacalcet was $61,705 per life-year and $79,562 per quality-adjusted life-year (QALY) gained using the covariate-adjusted ITT analysis. Probabilistic sensitivity analysis suggested a 73.2% chance of the ICER being below a willingness-to-pay threshold of $100,000. Treatment effects from unadjusted ITT analysis yielded an ICER of $115,876 per QALY. The model was most sensitive to the treatment effect on mortality.ConclusionsIn the unadjusted ITT analysis, cinacalcet does not represent a cost- effective use of health care resources when applying a willingness-to-pay threshold of $100,000 per QALY. When using the covariate-adjusted ITT treatment effect, which represents the least biased estimate, however, cinacalcet is a cost-effective therapy for patients with moderate-to-severe sHPT on hemodialysis.     

Cost-effectiveness analysis of a surveillance program to prevent hip dislocation in children with cerebral palsy

ObjectiveIn this study we conducted an economic evaluation of a surveillance programme to prevent hip dislocation in children with cerebral palsy.MethodWe developed a model that compared costs and health outcomes of children with cerebral palsy with and without a surveillance programme. Information from a number of sources was combined into a decision analytical model, primarily based on data from a comparative study with a 20-year follow-up. Effectiveness was measured using Quality-Adjusted Life Years (QALYs). The analysis took the perspective of the Spanish National Health Service. We undertook extensive sensitivity analyses including a probabilistic sensitivity analysis.ResultsThe surveillance programme led to higher QALYs and higher health care costs, with an estimated incremental cost per QALY gained of 12,282€. The results were robust to model assumptions. The probability that the programme was cost-effective was estimated to be over 80% at the threshold of 25.000€/QALY recommended in Spain.ConclusionThis study indicates that surveillance programmes to prevent hip dislocation in children with cerebral palsy are likely to be cost-effective.     

我国乳腺癌筛查卫生经济学研究的系统评价

目的 了解我国大陆地区乳腺癌筛查的卫生经济学评价进展。方法 系统检索PubMed、中国知网、万方数据知识服务平台和维普网1995年1月至2015年12月收录文献,对纳入研究基本信息、人群项目参与率及检出率、模型研究方法学、经济学评价方法及结果等信息进行摘录和比较,采用卫生经济学评价报告规范（CHEERS）评价报告质量（总分24分）。结果 共检索356篇文献,最终纳入13篇,均发表于近4年（2012-2015年）,其中11篇基于人群、3篇基于模型研究。筛查起始年龄为18～45岁,终止年龄均≥59岁;筛查技术包括临床检查、超声和钼靶单一或联合筛查。有7篇报道了研究角度,其中为政府等服务提供方5篇,社会角度2篇;仅有5篇研究进行了成本和（或）效果贴现。11篇成本-效果分析中,有9篇提供了评价指标检出1例乳腺癌的成本,为5.0～229.3（M=14.5）万元。以质量调整生命年（QALY）或伤残调整生命年（DALY）为指标的成本-效用分析仅4篇,相应增量成本效果比（ICER）为0.3万元～27.1万元（2015年我国人均GDP为4.9万元）。13篇文献平均得分14.5（9.5～21.0）分,总分24分,其中研究角度、贴现率、ICER及不确定性等维度得分较低。结论 我国大陆地区乳腺癌筛查的经济学研究尚处于起步阶段,尤其是模型研究;各研究间方法及结果可比性一般,报告质量有待加强。应从社会角度全面核算成本后对筛查项目开展以QALY或DALY为指标的成本-效用分析。     

Cost effectiveness of prostacyclins in pulmonary arterial hypertension

Background

Pulmonary arterial hypertension (PAH) is considered an orphan disease. Prostacyclins are the keystone for PAH treatment. Choosing between the three available prostacyclin therapies could be complicated because there are no comparison studies, so the final decision must be driven by factors such as efficacy, administration route, safety profile and economic aspects.

Objective

This study provides a cost-effectiveness and cost-utility comparison of initiating prostacyclin therapy with three different treatment alternatives (inhaled iloprost [ILO], intravenous epoprostenol [EPO] and subcutaneous treprostinil [TRE]) for patients with PAH. The goal of this work is to help physicians with their therapeutic decision-making.

Methods

A Markov model was built to simulate a patient cohort with class III PAH according to the classification of the New York Heart Association (NYHA). Four health states corresponding with the NYHA classes plus death were allowed for patients in the model. Changing the treatment was possible when patients worsened from functional class III to IV. The time horizon was 3 years, allowing patients to transition between health states on a 12-week cycle basis. The study perspective was that of the National Health System (NHS) [only direct medical costs were included]. Unitary costs were obtained from the Drug Catalogue and e-Salud Database in 2009 and are given in euros (?). Data on health resources and treatment pathways were informed by a four-member expert panel. Efficacy was obtained from pivotal clinical trials of ILO, EPO and TRE, the latter used in Spain as a foreign medication. Utilities for each health state were obtained from the literature. The final efficacy measure was life-years gained (LYG), and utilities were used to obtain quality-adjusted life-years (QALYs). Costs and effects were discounted at a 3% rate. To check for the robustness of the results, sensitivity analyses were performed.

Results

At the end of the 3 years, in the base case of the deterministic analysis, initiating prostacyclin therapy with iloprost was the less costly strategy (?132840), followed by treprostinil (?359 869) and epoprostenol (?429 775). Epoprostenol has shown the best efficacy results with 2.73 LYG and 1.78 QALY, followed by iloprost (2.69 LYG and 1.74 QALY) and treprostinil (2.69 LYG and 1.73 QALY). Incremental cost-effectiveness ratios (ICER) and cost-utility ratios (ICUR) of epoprostenol versus iloprost and treprostinil were much above the ?30 000 per LYG or QALY threshold commonly used in Spain. Iloprost was dominant compared with treprostinil. In the probabilistic analysis, epoprostenol, when compared with iloprost, was a dominant strategy in 15% of the simulations, but it was not a cost-effective option in 83% of the cases. When compared with treprostinil, epoprostenol was dominant in 43% of the simulations. Iloprost was dominant compared with treprostinil in 45% of the cases and it was a cost-effective alternative in 39% of the simulations.

Conclusions

Initiating prostacyclin treatment with iloprost in patients with PAH, functional class III of the NYHA, is the less costly alternative for the NHS in Spain, with a good efficacy profile when compared with the other alternatives.     

我国消除丙型肝炎的普通人群HCV检测策略的成本效果分析

目的 分析我国消除丙型肝炎（丙肝）的普通人群HCV检测策略的成本效果,明确最佳成本效果的HCV检测年龄。方法 运用TreeAge pro 2019软件构建决策树马尔科夫模型,以1年为周期,模拟10万名20~59岁各年龄组人群HCV检测和治疗结果,以全社会角度分析策略间比较的成本效果和效益。效果指标为增量成本效果比（ICER）,效益指标为净货币效益（NMB）,以我国2022年人均国内生产总值（85 698元）为意愿支付阈值。通过单因素敏感性分析和概率敏感性分析评估结果可靠性。结果 在20~59岁人群HCV检测有成本效果,在40~49岁年龄组进行HCV检测成本效果最佳。20~59岁年龄组人群HCV检测策略与未HCV检测策略比较,增量成本为161.24元/人,增量效用为0.003 6质量调整寿命年（QALYs）/人,ICER为45 197.26元/QALY,ICER小于意愿支付阈值,具有成本效果。各年龄组人群HCV检测策略与未HCV检测策略比较,ICER为42 055.06~53 249.43元/QALY,NMB为96.52~169.86元/人,其中40~49岁年龄组的ICER最低,NMB最高。单因素敏感性分析结果显示,贴现率、丙肝抗体（抗-HCV）检测成本、人群抗-HCV阳性率和直接抗病毒药物治疗成本对经济学评价影响较大,但改变参数取值,结论不变。概率敏感性分析结果表明模型分析结果稳定。结论 医疗机构探索动员20~59岁普通人群进行HCV检测具有较好的成本效果,以40~49岁年龄组人群的HCV检测成本效果最佳。在我国普通人群中实施HCV检测的“愿检尽检”策略,能降低人群丙肝疾病负担。