Value for Money in H1N1 Influenza: A Systematic Review of the Cost-Effectiveness of Pandemic Interventions

Background

The 2009 A/H1N1 influenza pandemic generated additional data and triggered new studies that opened debate over the optimal strategy for handling a pandemic. The lessons-learned documents from the World Health Organization show the need for a cost estimation of the pandemic response during the risk-assessment phase. Several years after the crisis, what conclusions can we draw from this field of research?

两地孕产妇产前检查方案的成本效果比较

目的 探讨比较两地孕产妇产前检查成本效果的方法。方法 分别调查两地孕产妇产前检查的费用,以及选用非低出生体重儿和非早产儿发生率作为效果的评价指标,得到成本效果比(CER)值,分别作两地CER值与产检次数的回归分析。结果 CER值与产检次数呈直线关系。结论 通过对两条回归直线的假设检验可对两地成本效果作比较。     

Economic Evaluation for Health Investments En Route to Universal Health Coverage: Cost-Benefit Analysis or Cost-Effectiveness Analysis?

Background

It is an unresolved issue as to whether cost-benefit analysis (CBA) or cost-effectiveness analysis (CEA) is the preferable analytical toolkit for use in health technology assessment (HTA). The distinction between the two and an expressed preference for CEA go back at least to 1980 in the USA and, most recently, a Harvard-based group has been reappraising the case for CBA.

Prioritizing Healthcare Interventions: A Comparison of Multicriteria Decision Analysis and Cost-Effectiveness Analysis

ObjectivesTo investigate the extent to which stated preferences for treatment criteria elicited using multicriteria decision analysis (MCDA) methods are consistent with the trade-offs (implicitly) applied in cost-effectiveness analysis (CEA), and the impact of any differences on the prioritization of treatments.MethodsWe used existing MCDA and CEA models developed to evaluate interventions for knee osteoarthritis in the New Zealand population. We established equivalent input parameters for each model, for the criteria “treatment effectiveness,” “cost,” “risk of serious harms,” and “risk of mild-to-moderate harms” across a comprehensive range of (hypothetical) interventions to produce a complete ranking of interventions from each model. We evaluated the consistency of these rankings between the 2 models and investigated any systematic differences between the (implied) weight placed on each criterion in determining rankings.ResultsThere was an overall moderate-to-strong correlation in intervention rankings between the MCDA and CEA models (Spearman correlation coefficient = 0.51). Nevertheless, there were systematic differences in the evaluation of trade-offs between intervention attributes and the resulting weights placed on each criterion. The CEA model placed lower weights on risks of harm and much greater weight on cost (at all accepted levels of willingness-to-pay per quality-adjusted life-year than did respondents to the MCDA survey.ConclusionsMCDA and CEA approaches to inform intervention prioritization may give systematically different results, even when considering the same criteria and input data. These differences should be considered when designing and interpreting such studies to inform treatment prioritization decisions.     

Assessing the Impact of Censoring of Costs and Effects on Health-Care Decision-Making: an Example Using the Atrial Fibrillation Follow-up Investigation of Rhythm Management (AFFIRM) Study

Objectives:  Losses to follow-up and administrative censoring can cloud the interpretation of trial-based economic evaluations. A number of investigators have examined the impact of different levels of adjustment for censoring, including nonadjustment, adjustment of effects only, and adjustment for both costs and effects. Nevertheless, there is a lack of research on the impact of censoring on decision-making. The objective of this study was to estimate the impact of adjustment for censoring on the interpretation of cost-effectiveness results and expected value of perfect information (EVPI), using a trial-based analysis that compared rate- and rhythm-control treatments for persons with atrial fibrillation.
Methods:  Three different levels of adjustment for censoring were examined: no censoring of cost and effects, censoring of effects only, and censoring of both costs and effects. In each case, bootstrapping was used to estimate the uncertainty incosts and effects, and the EVPI was calculated to determine the potential worth of further research.
Results:  Censoring did not impact the adoption decision. Nevertheless, this was not the case for the decision uncertainty or the EVPI. For a threshold of $50,000 per life-year, the EVPI varied between $626,000 (partial censoring) to $117 million (full censoring) for the eligible US population.
Conclusions:  The level of adjustment for censoring in trial-based cost-effectiveness analyses can impact on the decisions to fund a new technology and to devote resources for further research. Only when censoring is taken into account for both costs and effects are these decisions appropriately addressed.     

Alternative Approaches to Quality-Adjusted Life-Year Estimation Within Standard Cost-Effectiveness Models: Literature Review,Feasibility Assessment,and Impact Evaluation

ObjectivesThe quality-adjusted life-year (QALY) has been long debated, but alternative estimation approaches have not been comprehensively evaluated. Our objective was to identify alternatives, characterize them by implementation feasibility, and evaluate the impact of implementing feasible options in cost-effectiveness models developed for the Institute for Clinical and Economic Review reports.MethodsWe conducted a literature review combining keywords relating to QALYs, methodology alternatives, and cost-effectiveness in PubMed, EconLit, Web of Science, and MEDLINE. Articles that discussed alternatives to the conventional QALY were included. Alternatives were characterized by type, data availability, calculation burden, and overall implementation feasibility. The subset of feasible alternatives, that is, sufficient data and methodology compatible with incorporation into common modeling approaches, were evaluated according to impact on incremental QALYs, incremental net monetary benefit (iNMB), intervention rankings, and proportion of interventions with a positive iNMB.ResultsWe identified 28 articles discussing 9 alternatives. Feasible alternatives were using patient preference (PP) data; equity weighting according to baseline utility, fair innings, or proportional QALY shortfall; and the equal value of life-years-gained approach. All alternatives affected the incremental QALY and iNMB outcomes, rankings, and proportion of interventions with a positive iNMB. The PP alternative had the largest and most consistent impact. The PP impact on the proportion of interventions with a positive iNMB, was in the negative direction.ConclusionsOur work is the first comprehensive evaluation of proposed alternatives to the conventional QALY. We found robust literature but few options that were feasible to be implemented in current healthcare decision-making processes.     

Would a Large tPA Trial for Those 4.5 to 6.0 Hours from Stroke Onset Be Good Value for Information?

Objectives

To quantify the potential value of new research in patients treated with thrombolytic treatment (tissue-type plasminogen activator [tPA]) in the 4.5- to 6.0-hour time window after stroke onset and to determine the optimal size of a future trial using value of information analysis.

与临床试验平行的经济学评价设计面临的挑战

目的:探讨临床试验同期平行进行的卫生经济学评价(以下简称平行研究)在数据收集和设计上可能出现的问题及所遇到的质疑,以及可能的解决方法。方法:回顾平行研究的方法学文献以及经选择的经验类研究文章。结果:平行研究在某些方面优于其他药物经济学评价方法,在(临床)试验过程中同时收集数据而不需要专门开展独立的经济学研究。但在平行研究的设计及数据收集中仍存在许多挑战和争论。结论:平行研究仍是测量医学干预在经济上影响的适当方式。     

A Transparent and Consistent Approach to Assess US Outpatient Drug Costs for Use in Cost-Effectiveness Analyses

Background

Assessment of drug costs for cost-effectiveness analyses (CEAs) in the United States is not straightforward because the prices paid for drugs are not publicly available and differ between payers. CEAs have relied on list prices that do not reflect the rebates and discounts known to be associated with these purchases.

Toward a Broader Concept of Value: Identifying and Defining Elements for an Expanded Cost-Effectiveness Analysis

This commentary identifies and defines potentially useful expansions to traditional cost-effectiveness analysis as often used in health technology assessment. Since the seminal 1977 article by Weinstein and Stason, the recommended approach has been the use of the incremental cost-effectiveness ratio based on the metric of the cost per quality-adjusted life-year gained, allowing comparisons across different technologies. An expanded framework, incorporating a wider range of the elements of value, is proposed. In addition to the core value drivers of health gain and other health system cost savings (if any), we propose adding other less recognized elements related to the value of knowing and informational externalities. We describe each of five factors related to the value of knowing: 1) a reduction in uncertainty, reflecting the benefit of a companion diagnostic increasing the certainty of a patient?s response to a medicine; 2) insurance value related to greater peace of mind due to protection against catastrophic health and financial loss; 3) the value of hope for a "cure," leading individuals to become risk seekers in some circumstances; 4) real option value due to life extension opening possibilities for individuals to benefit from future innovation; and 5) spillovers or externalities arising from benefits of scientific advances that cannot be entirely appropriated by those making the advances. Further thought and research are needed on how best to measure and integrate these elements into an incremental value framework and on coverage and pricing decisions.     

Advancing Value Assessment in the United States: A Multistakeholder Perspective

Rising costs without perceived proportional improvements in quality and outcomes have motivated fundamental shifts in health care delivery and payment to achieve better value. Aligned with these efforts, several value assessment frameworks have been introduced recently to help providers, patients, and payers better understand the potential value of drugs and other interventions and make informed decisions about their use. Given their early stage of development, it is imperative to evaluate these efforts on an ongoing basis to identify how best to support and improve them moving forward. This article provides a multistakeholder perspective on the key limitations and opportunities posed by the current value assessment frameworks and areas of and actions for improvement. In particular, we outline 10 fundamental guiding principles and associated strategies that should be considered in subsequent iterations of the existing frameworks or by emerging initiatives in the future. Although value assessment frameworks may not be able to meet all the needs and preferences of stakeholders, we contend that there are common elements and potential next steps that can be supported to advance value assessment in the United States.     

Cost-Effectiveness Analysis of Smoking Cessation Interventions in the United Kingdom Accounting for Major Neuropsychiatric Adverse Events

ObjectivesSmoking is a leading cause of death worldwide. Cessation aids include varenicline, bupropion, nicotine replacement therapy (NRT), and e-cigarettes at various doses (low, standard and high) and used alone or in combination with each other. Previous cost-effectiveness analyses have not fully accounted for adverse effects nor compared all cessation aids. The objective was to determine the relative cost-effectiveness of cessation aids in the United Kingdom.MethodsAn established Markov cohort model was adapted to incorporate health outcomes and costs due to depression and self-harm associated with cessation aids, alongside other health events. Relative efficacy in terms of abstinence and major adverse neuropsychiatric events was informed by a systematic review and network meta-analysis. Base case results are reported for UK-licensed interventions only. Two sensitivity analyses are reported, one including unlicensed interventions and another comparing all cessation aids but removing the impact of depression and self-harm. The sensitivity of conclusions to model inputs was assessed by calculating the expected value of partial perfect information.ResultsWhen limited to UK-licensed interventions, varenicline standard-dose and NRT standard-dose were most cost-effective. Including unlicensed interventions, e-cigarette low-dose appeared most cost-effective followed by varenicline standard-dose + bupropion standard-dose combined. When the impact of depression and self-harm was excluded, varenicline standard-dose + NRT standard-dose was most cost-effective, followed by varenicline low-dose + NRT standard-dose.ConclusionAlthough found to be most cost-effective, combined therapy is currently unlicensed in the United Kingdom and the safety of e-cigarettes remains uncertain. The value-of-information analysis suggested researchers should continue to investigate the long-term effectiveness and safety outcomes of e-cigarettes in studies with active comparators.     

Evaluating Frameworks That Provide Value Measures for Health Care Interventions

The recent acceleration of scientific discovery has led to greater choices in health care. New technologies, diagnostic tests, and pharmaceuticals have widely varying impact on patients and populations in terms of benefits, toxicities, and costs, stimulating a resurgence of interest in the creation of frameworks intended to measure value in health. Many of these are offered by providers and/or advocacy organizations with expertise and interest in specific diseases (e.g., cancer and heart disease). To help assess the utility of and the potential biases embedded in these frameworks, we created an evaluation taxonomy with seven basic components: 1) define the purpose; 2) detail the conceptual approach, including perspectives, methods for obtaining preferences of decision makers (e.g., patients), and ability to incorporate multiple dimensions of value; 3) discuss inclusions and exclusions of elements included in the framework, and whether the framework assumes clinical intervention or offers alternatives such as palliative care or watchful waiting; 4) evaluate data sources and their scientific validity; 5) assess the intervention’s effect on total costs of treating a defined population; 6) analyze how uncertainty is incorporated; and 7) illuminate possible conflicts of interest among those creating the framework. We apply the taxonomy to four representative value frameworks recently published by professional organizations focused on treatment of cancer and heart disease and on vaccine use. We conclude that each of these efforts has strengths and weaknesses when evaluated using our taxonomy, and suggest pathways to enhance the utility of value-assessing frameworks for policy and clinical decision making.     

Economic Evaluations of Pathology Tests, 2010-2015: A Scoping Review

Background

Concerns about pathology testing such as the value provided by new tests and the potential for inappropriate utilization have led to a greater need to assess costs and benefits. Economic evaluations are a formal method of analyzing costs and benefits, yet for pathology tests, questions remain about the scope and quality of the economic evidence.

Use of Checklists in Reviews of Health Economic Evaluations, 2010 to 2018

Objective

It is useful for reviewers of economic evaluations to assess quality in a manner that is consistent and comprehensive. Checklists can allow this, but there are concerns about their reliability and how they are used in practice. We aimed to describe how checklists have been used in systematic reviews of health economic evaluations.

中医与西医门诊治疗同病种费用的经济学评价

通过现场回顾性调查,收集基层医疗服务机构中医与西医门诊治疗同病种的费用以及治疗效果的数据资料,采用成本效果分析方法,对病种相同、病情相似的患者分别采用纯中医或纯西医治疗,获得相同治疗效果的费用进行了比较分析,通过实证研究佐证中医服务具有＂廉＂的特点,从而阐明中医在基层医疗服务机构发展的优势与意义。     

Good Research Practices for Measuring Drug Costs in Cost-Effectiveness Analyses: A Managed Care Perspective: The ISPOR Drug Cost Task Force Report—Part III

Objectives:  The objective of this report is to provide guidance and recommendations on how drug costs should be measured for cost-effectiveness analyses conducted from the perspective of a managed care organization (MCO).
Methods:  The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force on Good Research Practices—Use of Drug Costs for Cost Effectiveness Analysis (DCTF) was appointed by the ISPOR Board of Directors. Members were experienced developers or users of CEA models. The DCTF met to develop core assumptions and an outline before preparing a draft report. They solicited comments on drafts from external reviewers and from the ISPOR membership at ISPOR meetings and via the ISPOR Web site.
Results:  The cost of a drug to an MCO equals the amount it pays to the dispenser for the drug's ingredient cost and dispensing fee minus the patient copay and any rebates paid by the drug's manufacturer. The amount that an MCO reimburses for each of these components can differ substantially across a number of factors that include type of drug (single vs. multisource), dispensing site (retail vs. mail order), and site of administration (self-administered vs. physician's office). Accurately estimating the value of cost components is difficult because they are determined by proprietary and confidential contracts.
Conclusion:  Estimates of drug cost from the MCO perspective should include amounts paid for medication ingredients and dispensing fees, and net out copays, rebates, and other drug price reductions. Because of the evolving nature of drug pricing, ISPOR should publish a Web site where current DCTF costing recommendations are updated as new information becomes available.     

经济分析、资本诉求与国家药物政策研究：一个理论框架

市场价值不同于道德或者伦理价值,当我们致力于研究国家药物政策时,应当清醒地意识到,尽管从总体目标来看国家药物政策本身体现了社会认可的公平性以及社会伦理的基本观念,但是,其实际的制订与实施很可能是一次又一次的利益博弈,不同的利益群体所认可的价值存在着根本性的差异。作为一种制度安排,国家药物政策的制订及其实施必定涉及到经济利益。制药企业是经济利益最主要的关联方,对于制药企业而言,国家药物政策中的经济目标和可能引致的经济后果是其所唯一关注的,并在国家药物政策从制订到实施的不同层级中寻求最大化其经济利益的可能。