Decision-analytic modelling

  • 文章类型: Systematic Review
    目的:评估心血管疾病(CVD)的干预措施需要评估其效用。我们旨在1)系统地审查自2013年以来发布的CVD的效用估计,2)严格评估英国相关的估计并计算相应的基线效用乘数。
    方法:我们使用CVD和效用项搜索了MEDLINE和Embase(2021年4月22日)。我们筛选了主要研究的结果,这些研究报告了有心力衰竭经历的人的效用分布,心肌梗塞,外周动脉疾病,稳定型心绞痛,中风,短暂性脑缺血发作,或者不稳定型心绞痛.我们从包括的研究中提取了特征。对于基于EuroQoL5维(EQ-5D)度量的英国估计,我们评估了偏差风险和对决策分析模型的适用性,适当的合并臂/时间点,并使用没有CVD的年龄和性别匹配人群的预测效用来估计基线效用乘数。我们从具有低偏倚风险的直接适用研究中寻求效用来源,在我们的基本案例模型中优先考虑严重性排序的合理性,并在敏感性分析中确定最高的人群。
    结果:确定的403项研究大多数使用EQ-5D(n=217),评估最多的经济合作与发展组织(n=262)。虽然措施和国家差异很大。使用EQ-5D(n=29)的英国研究为每种类型的CVD产生了非常异质的基线效用乘数,排除荟萃分析并暗示不同的可能严重程度顺序。我们可以找到提供合理的公用事业订购的来源,同时充分代表健康状态。
    结论:我们对国际CVD效用估计进行了分类,并计算了英国相关的基线效用乘数。建模人员应考虑未报告的异构源,比如人口差异,从评论中选择效用证据时。
    结论:发表的系统综述总结了截至2013年发表的与心血管疾病相关的效用估计。我们1)回顾了自2013年以来发表的7种心血管疾病的效用估计,2)严格评估了英国相关研究,和3)估计每种心血管疾病对基线效用的影响。我们的综述1)为7种类型的心血管疾病推荐了一组一致且可靠的基线效用乘数,2)为寻求自身背景的效用证据的研究人员提供了系统识别的参考信息。
    OBJECTIVE: Evaluating interventions for cardiovascular disease (CVD) requires estimates of its effect on utility. We aimed to 1) systematically review utility estimates for CVDs published since 2013 and 2) critically appraise UK-relevant estimates and calculate corresponding baseline utility multipliers.
    METHODS: We searched MEDLINE and Embase (April 22, 2021) using CVD and utility terms. We screened results for primary studies reporting utility distributions for people with experience of heart failure, myocardial infarction, peripheral arterial disease, stable angina, stroke, transient ischemic attack, or unstable angina. We extracted characteristics from studies included. For UK estimates based on the EuroQoL 5-dimension (EQ-5D) measure, we assessed risk of bias and applicability to a decision-analytic model, pooled arms/time points as appropriate, and estimated baseline utility multipliers using predicted utility for age- and sex- matched populations without CVD. We sought utility sources from directly applicable studies with low risk of bias, prioritizing plausibility of severity ordering in our base-case model and highest population ascertainment in a sensitivity analysis.
    RESULTS: Most of the 403 studies identified used EQ-5D (n = 217) and most assessed Organisation for Economic Co-operation and Development populations (n = 262), although measures and countries varied widely. UK studies using EQ-5D (n = 29) produced very heterogeneous baseline utility multipliers for each type of CVD, precluding meta-analysis and implying different possible severity orderings. We could find sources that provided a plausible ordering of utilities while adequately representing health states.
    CONCLUSIONS: We cataloged international CVD utility estimates and calculated UK-relevant baseline utility multipliers. Modelers should consider unreported sources of heterogeneity, such as population differences, when selecting utility evidence from reviews.
    CONCLUSIONS: Published systematic reviews have summarized estimates of utility associated with cardiovascular disease published up to 2013.We 1) reviewed utility estimates for 7 types of cardiovascular disease published since 2013, 2) critically appraised UK-relevant studies, and 3) estimated the effect of each cardiovascular disease on baseline utility.Our review 1) recommends a consistent and reliable set of baseline utility multipliers for 7 types of cardiovascular disease and 2) provides systematically identified reference information for researchers seeking utility evidence for their own context.
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  • 文章类型: Journal Article
    A global plan has been set to end human deaths from dog-mediated rabies by 2030 (\"Zero-by-30\"), but whether it could be achieved in some countries, such as China, remains unclear. Although elimination strategies through post-exposure prophylaxis (PEP) use, dog vaccination, and patient risk assessments with integrated bite case management (IBCM) were proposed to be cost-effective, evidence is still lacking in China. We aim to evaluate the future burdens of dog-mediated human rabies deaths in the next decade and provide quantitative evidence on the cost-effectiveness of different rabies-control strategies in China.
    Based on data from China\'s national human rabies surveillance system, we used decision-analytic modelling to estimate dog-mediated human rabies death trends in China till 2035. We simulated and compared the expected consequences and costs of different combination strategies of the status quo, improved access to PEP, mass dog vaccination, and use of IBCM.
    The predicted human rabies deaths in 2030 in China will be 308 (95%UI: 214-411) and remain stable in the next decade under the status quo. The strategy of improved PEP access alone could only decrease deaths to 212 (95%UI: 147-284) in 2028, remaining unchanged till 2035. In contrast, scaling up dog vaccination to coverage of 70% could eliminate rabies deaths by 2033 and prevent approximately 3,265 (95%UI: 2,477-3,687) extra deaths compared to the status quo during 2024-2035. Moreover, with the addition of IBCM, the \"One Health\" approach through mass dog vaccination could avoid unnecessary PEP use and substantially reduce total cost from 12.53 (95%UI: 11.71-13.34) to 8.73 (95%UI: 8.09-9.85) billion US dollars. Even if increasing the total costs of IBCM from 100 thousand to 652.10 million US dollars during 2024-2035, the combined strategy of mass dog vaccination and use of IBCM will still dominate, suggesting the robustness of our results.
    The combined strategy of mass dog vaccination and IBCM requires collaboration between health and livestock/veterinary sectors, and it could eliminate Chinese rabies deaths as early as 2033, with more deaths averted and less cost, indicating that adding IBCM could reduce unnecessary use of PEP and make the \"One Health\" rabies-control strategy most cost-effective.
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  • 文章类型: Journal Article
    目的:评价基于决策分析模型的老年患者(≥70岁)原发性乳腺癌(PBC)治疗成本效益分析的证据来源和方法。
    方法:两个电子数据库(OvidMedline,OvidEMBASE)进行了搜索(开始至2021年9月5日),以确定基于模型的PBC老年女性治疗方法的全面经济评估,作为其基本病例目标人群或年龄亚组分析的一部分。评估四种类型的输入参数的数据来源和方法,包括与健康相关的生活质量(HRQoL);自然史;治疗效果;提取并评估了资源使用情况。质量评估是通过参考综合卫生经济评价报告标准完成的。
    结果:纳入了7项基于模型的经济学评价(老年患者作为基础病例(n=3)或亚组(n=4)分析的一部分)。来自年轻患者(<70岁)的数据经常被用来估计输入参数。采用不同的方法来调整老年人口的这些估计(HRQoL:无效乘数,添加剂效用递减;自然史:绝对值的校准,单向敏感性分析;治疗效果:观察数据分析,特定年龄的行为参数,合理的情景分析;资源使用:匹配的对照观测数据分析,取决于年龄的后续费用)。
    结论:改善老年PBC患者的估计输入参数将改善成本效益的估计,决策不确定性,以及进一步研究的价值。这篇综述中报告的方法可以为未来的成本效益分析提供信息,以克服该人群的数据挑战。更好地了解这些患者的治疗价值将改善人群健康结果,临床决策,和资源分配决策。
    OBJECTIVE: To appraise the sources of evidence and methods to estimate input parameter values in decision-analytic model-based cost-effectiveness analyses of treatments for primary breast cancer (PBC) in older patients (≥ 70 years old).
    METHODS: Two electronic databases (Ovid Medline, Ovid EMBASE) were searched (inception until 5 September-2021) to identify model-based full economic evaluations of treatments for older women with PBC as part of their base-case target population or age-subgroup analysis. Data sources and methods to estimate four types of input parameters including health-related quality of life (HRQoL); natural history; treatment effect; resource use were extracted and appraised. Quality assessment was completed by reference to the Consolidated Health Economic Evaluation Reporting Standards.
    RESULTS: Seven model-based economic evaluations were included (older patients as part of their base-case (n = 3) or subgroup (n = 4) analysis). Data from younger patients (< 70 years) were used frequently to estimate input parameters. Different methods were adopted to adjust these estimates for an older population (HRQoL: disutility multipliers, additive utility decrements; Natural history: calibration of absolute values, one-way sensitivity analyses; Treatment effect: observational data analysis, age-specific behavioural parameters, plausible scenario analyses; Resource use: matched control observational data analysis, age-dependent follow-up costs).
    CONCLUSIONS: Improving estimated input parameters for older PBC patients will improve estimates of cost-effectiveness, decision uncertainty, and the value of further research. The methods reported in this review can inform future cost-effectiveness analyses to overcome data challenges for this population. A better understanding of the value of treatments for these patients will improve population health outcomes, clinical decision-making, and resource allocation decisions.
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  • 文章类型: Journal Article
    母乳喂养与母亲和婴儿的健康益处以及卫生服务的成本节约有关。由于各种原因,包括文化障碍,英国的母乳喂养率很低,对启动和维持母乳喂养的支持不足,缺乏信息,或者选择不母乳喂养。已经制定了旨在提高母乳喂养率的教育和支持干预措施。这项研究的目的是评估这种干预措施对妇女的成本效益,在产前或出生后的前8周内开始,旨在提高母乳喂养率,在英国。
    从英格兰健康和个人社会服务的角度,构建了一个决策分析模型,以比较母乳喂养干预措施的成本和质量调整寿命年(QALYs)。干预效果和母乳喂养益处的数据来自系统评价。其他模型输入参数是从公开来源获得的,补充专家意见。
    标准护理与标准护理相比,模型干预措施的增量成本效益比(ICER)为51,946英镑/QALY,这表明,根据英国国家健康与护理卓越研究所(NICE)的标准,该干预措施不具成本效益。敏感性分析表明,干预措施的成本效果随着干预效果的提高和干预成本的降低而提高。在基本情况下(出生后16-26周母乳喂养率增加19%),如果每位接受干预的女性的成本为≈40-45英镑,则干预是具有成本效益的(<20,000英镑/QALY).按基本情况计算(84英镑),如果该干预措施使母乳喂养率至少提高35-40%,则具有成本效益.
    在英国,根据NICE标准,现有的母乳喂养干预措施似乎没有成本效益。与目前可用的干预措施相比,未来的母乳喂养干预措施需要具有更高的有效性或更低的成本,以便具有成本效益。公共卫生和其他社会干预措施,促进和支持母乳喂养可能是提高英国母乳喂养率的关键.
    Breastfeeding is associated with health benefits to mothers and babies and cost-savings to the health service. Breastfeeding rates in the UK are low for various reasons including cultural barriers, inadequate support to initiate and sustain breastfeeding, lack of information, or choice not to breastfeed. Education and support interventions have been developed aiming at promoting breastfeeding rates. The objective of this study was to assess the cost-effectiveness of such interventions for women, initiated antenatally or in the first 8 weeks postnatally, aiming at improving breastfeeding rates, in the UK.
    A decision-analytic model was constructed to compare costs and quality-adjusted life-years (QALYs) of a breastfeeding intervention from the perspective of health and personal social services in England. Data on intervention effectiveness and the benefits of breastfeeding were derived from systematic reviews. Other model input parameters were obtained from published sources, supplemented by expert opinion.
    The incremental cost-effectiveness ratio (ICER) of the modelled intervention added on standard care versus standard care was £51,946/QALY, suggesting that the intervention is not cost-effective under National Institute for Health and Care Excellence (NICE) criteria in England. Sensitivity analysis suggested that the cost-effectiveness of the intervention improved as its effectiveness increased and intervention cost decreased. At the base-case effect (increase in breastfeeding rates 16-26 weeks after birth by 19%), the intervention was cost-effective (<£20,000/QALY) if its cost per woman receiving the intervention became ≈£40-£45. At the base-case cost (£84), the intervention was cost-effective if it increased breastfeeding rates by at least 35-40%.
    Available breastfeeding interventions do not appear to be cost-effective under NICE criteria in England. Future breastfeeding interventions need to have higher effectiveness or lower cost compared with currently available interventions in order to become cost-effective. Public health and other societal interventions that protect, promote and support breastfeeding may be key in improving breastfeeding rates in the UK.
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  • 文章类型: Journal Article
    PTSD in youth may lead to long-lasting psychological implications, educational difficulties and increased healthcare costs. Psychological interventions have been shown to be effective in its management. The objective of this study was to assess the cost-effectiveness of a range of psychological interventions for children and young people with PTSD.
    A decision-analytic model was constructed to compare costs and quality-adjusted life years (QALYs) of 10 psychological interventions and no treatment for children and young people with PTSD, from the perspective of the National Health Service and personal social services in England. Effectiveness data were derived from a systematic review and network meta-analysis. Other model input parameters were based on published sources, supplemented by expert opinion.
    Cognitive therapy for PTSD, a form of individual trauma-focused cognitive behavioural therapy (TF-CBT), appeared to be the most cost-effective intervention for children and young people with PTSD (with a probability of .78 amongst the 11 evaluated options at a cost-effectiveness threshold of £20,000/QALY), followed by narrative exposure (another form of individual TF-CBT), play therapy, and other forms of individual TF-CBT. After excluding cognitive therapy from the analysis, narrative exposure appeared to be the most cost-effective option with a .40 probability of being cost-effective amongst the remaining 10 options. EMDR, parent training and group TF-CBT occupied middle cost-effectiveness rankings. Family therapy and supportive counselling were less cost-effective than other active interventions. There was limited evidence for some interventions, in particular cognitive therapy for PTSD and parent training.
    Individual forms of TF-CBT and, to a lesser degree, play therapy appear to be cost-effective in the treatment of children and young people with PTSD. Family therapy and supportive counselling are unlikely to be cost-effective relative to other interventions. There is a need for well-conducted studies that examine the long-term clinical and cost-effectiveness of a range of psychological treatments for children and young people with PTSD.
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  • 文章类型: Journal Article
    背景:当智力残疾的人变老时,国际上出现的证据表明,这一特定的老龄化人口的健康需求基本上未得到满足。一些国家已经实施了健康检查,以解决这些健康不平等问题。由于测量生活质量结果的挑战,评估的重点是测量过程结果。此外,成本效益目前未知。作为针对该人群的国家指南的一部分,我们试图探索英格兰年度健康检查的可能成本效益。
    方法:使用决策分析马尔可夫模型来估计策略的成本效益,为智力残疾的老年人提供健康检查,与标准护理相比。我们采取的方法是探索性的。针对选定的一系列健康状况开发了单独的模型,具有预期的高经济影响,并且有足够的证据可用于建模。在每个模型中,假设队列从40年开始随访。年龄直到死亡。结果衡量标准是获得的每质量调整生命年成本(QALY)。计算了增量成本效益比(ICER)。从卫生提供者的角度评估了成本,并在2016年GBP表示。成本和QALY折现为3.5%。我们进行了概率敏感性分析。来自已发表研究的数据以及专家意见的参数。
    结果:健康检查导致平均QALY收益为0.074(95%CI0.072至0.119);平均增量成本为4787英镑(CI95%4773至5017)。对于每QALY30,000英镑的门槛,健康检查不划算(平均ICER£85,632;95%CI82,762至131,944)。为了使健康检查具有成本效益,干预费用需要从每年258英镑减少到100英镑以下。
    结论:虽然研究结果需要谨慎考虑,因为该模型是探索性的,因为它是基于克服证据差距的假设,他们认为,卫生系统为弱势群体提供护理的方式可能需要重新审查。这项工作是作为国家指南的一部分进行的,并为实现更平等的医疗保健规定的制度改革提供了建议。
    BACKGROUND: Whilst people with intellectual disability grow older, evidence has emerged internationally about the largely unmet health needs of this specific ageing population. Health checks have been implemented in some countries to address those health inequalities. Evaluations have focused on measuring process outcomes due to challenges measuring quality of life outcomes. In addition, the cost-effectiveness is currently unknown. As part of a national guideline for this population we sought to explore the likely cost-effectiveness of annual health checks in England.
    METHODS: Decision-analytical Markov modelling was used to estimate the cost-effectiveness of a strategy, in which health checks were provided for older people with intellectual disability, when compared with standard care. The approach we took was explorative. Individual models were developed for a selected range of health conditions, which had an expected high economic impact and for which sufficient evidence was available for the modelling. In each of the models, hypothetical cohorts were followed from 40 yrs. of age until death. The outcome measure was cost per quality-adjusted life-year (QALY) gained. Incremental cost-effectiveness ratios (ICER) were calculated. Costs were assessed from a health provider perspective and expressed in 2016 GBP. Costs and QALYs were discounted at 3.5%. We carried out probabilistic sensitivity analysis. Data from published studies as well as expert opinion informed parameters.
    RESULTS: Health checks led to a mean QALY gain of 0.074 (95% CI 0.072 to 0.119); and mean incremental costs of £4787 (CI 95% 4773 to 5017). For a threshold of £30,000 per QALY, health checks were not cost-effective (mean ICER £85,632; 95% CI 82,762 to 131,944). Costs of intervention needed to reduce from £258 to under £100 per year in order for health checks to be cost-effective.
    CONCLUSIONS: Whilst findings need to be considered with caution as the model was exploratory in that it was based on assumptions to overcome evidence gaps, they suggest that the way health systems deliver care for vulnerable populations might need to be re-examined. The work was carried out as part of a national guideline and informed recommendations about system changes to achieve more equal health care provisions.
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  • 文章类型: Journal Article
    收益时间表示对干预措施的收益何时超过成本的估计。对于具有递延收益但前期危害的干预措施,它对于利益-损害决策特别有用。这项研究的目的是扩大收益时间的应用范围,并提供一个在决策分析模型中使用的示例。
    根据估计的10年心血管风险的不同水平,三个临床相关的患者小插曲(10%,15%,20%)被开发。采用健康服务观点和生命周期的现有状态转移马尔可夫模型进行了调整,以包括与持续使用他汀类药物相关的3个直接治疗无效性(DTD)水平:0.005、0.01和0.015。对于每个小插图和DTD,我们计算了一系列输出,包括包含和不包括医疗保健成本的支付时间。
    对于10%的10年心血管风险(插图1)与低水平的DTD(0.005),不包括成本时的收益时间为8.5年,包括成本时的收益时间为16年。随着心血管疾病的基线风险增加,支付时间缩短。对于15%的心血管风险(插图2)和低水平的DTD,回报时间是5.5年和9.5年,分别。对于20%的心血管风险(插图3),回报时间为4.2年和7.2年,分别。对于每个小插图的更高水平的DTD,回报时间延长了,在某些情况下,干预从未得到回报,导致患者预期的净伤害。
    这项研究显示了如何将收益时间应用于现有的决策分析模型,并用于补充现有措施以指导医疗保健决策。
    The payoff time represents an estimate of when the benefits of an intervention outweigh the costs. It is particularly useful for benefit-harm decision making for interventions that have deferred benefits but upfront harms. The aim of this study was to expand the application of the payoff time and provide an example of its use within a decision-analytic model.
    Three clinically relevant patient vignettes based on varying levels of estimated 10-year cardiovascular risk (10%, 15%, 20%) were developed. An existing state-transition Markov model taking a health service perspective and a life-time horizon was adapted to include 3 levels of direct treatment disutility (DTD) associated with ongoing statin use: 0.005, 0.01, and 0.015. For each vignette and DTD we calculated a range of outputs including the payoff time inclusive and exclusive of healthcare costs.
    For a 10% 10-year cardiovascular risk (vignette 1) with low-levels of DTD (0.005), the payoff time was 8.5 years when costs were excluded and 16 years when costs were included. As the baseline risk of cardiovascular increased, the payoff time shortened. For a 15% cardiovascular risk (vignette 2) and for a low-level of DTD, the payoff time was 5.5 years and 9.5 years, respectively. For a 20% cardiovascular risk (vignette 3), the payoff time was 4.2 and 7.2 years, respectively. For higher levels of DTDs for each vignette, the payoff time lengthened, and in some instances the intervention never paid off, leading to an expected net harm for patients.
    This study has shown how the payoff time can be readily applied to an existing decision-analytic model and be used to complement existing measures to guide healthcare decision making.
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  • 文章类型: Journal Article
    Our aim was to adapt the traditional framework for expected net benefit of sampling (ENBS) to be more compatible with drug development trials from the pharmaceutical perspective. We modify the traditional framework for conducting ENBS and assume that the price of the drug is conditional on the trial outcomes. We use a value-based pricing (VBP) criterion to determine price conditional on trial data using Bayesian updating of cost-effectiveness (CE) model parameters. We assume that there is a threshold price below which the company would not market the new intervention. We present a case study in which a phase III trial sample size and trial duration are varied. For each trial design, we sampled 10,000 trial outcomes and estimated VBP using a CE model. The expected commercial net benefit is calculated as the expected profits minus the trial costs. A clinical trial with shorter follow-up, and larger sample size, generated the greatest expected commercial net benefit. Increasing the duration of follow-up had a modest impact on profit forecasts. Expected net benefit of sampling can be adapted to value clinical trials in the pharmaceutical industry to optimise the expected commercial net benefit. However, the analyses can be very time consuming for complex CE models.
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