Presenting Evidence and Summary Measures to Best Inform Societal Decisions When Comparing Multiple Strategies
Presenting Evidence and Summary Measures to Best Inform Societal Decisions When Comparing Multiple Strategies
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DOI：
10.2165/11587100-000000000-00000
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发表时间：
2011-01-01
影响因子：
4.4
通讯作者：
Willan, Andrew R.
中科院分区：
文献类型：
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作者：
Eckermann, Simon;Willan, Andrew R.
卫生技术评估（HTA）中的多种策略比较变得越来越重要，有多种替代治疗措施，治疗组合以及诊断和基因检测替代方案。在不确定性条件下，比较两种以上战略的增量成本、效果和成本效益，在概念上和实践上都与两种战略的比较大不相同，在两种战略中，所有证据都可以总结为增量成本效益平面上的单一双变量分布。在卫生技术评估中比较多种策略的替代方法已经开发出来：（i）在成本-负效用平面上呈现成本和效果;（ii）用多种策略的成本-效果可接受性（CEA）和预期净损失（ENL）曲线和边界总结证据。然而，对于分析师和决策者来说，关键问题仍然是如何在多种策略中最好地使用这些技术，以（i）在提供证据时告知临床和成本推断，以及（ii）总结成本效益的证据，为社会报销决策提供信息，其中偏好可能是风险中性或箭头下的风险厌恶-林德定理。我们批判性地考虑如何跨多种策略的证据可以最好地呈现和总结，以告知推理和社会报销决策，鉴于目前可用的方法。在这个过程中，我们提出了一些重要的原始发现。首先，在提出多个策略的证据，成本和效果的联合分布在成本负效用平面与相关的灵活的比较不同的复制成本和效果轴，确保充分的成本和效果的推断。这样的推论通常是混淆的成本效益平面上的比较相对于一个固定的原点和axes.Second，在总结的证据，风险中性的社会决策，ENL曲线和边界的CEA前沿直接提出的差异，预期净效益（ENB）的优势。CEA边界，而确定的战略，最大化ENB，只提出了他们的概率最大化净效益（NB），因此，无法解释为什么战略最大化ENB在任何给定的阈值。贸易-最大化ENB的策略与具有最大化NB的更高概率的其他潜在最优策略之间的权衡应在离散阈值在哪里出现。然而，概率告知这些权衡和相关的离散阈值区域应来自双边CEA曲线，以防止混杂的其他战略固有的多策略CEA curve.Based上这些发现，一系列的建议，最好的呈现和总结成本效益的证据，报销决策时，比较多个战略，这与比较两种策略的建议形成对比。联合研究和报销决定的影响也进行了讨论。
Multiple strategy comparisons in health technology assessment (HTA) are becoming increasingly important, with multiple alternative therapeutic actions, combinations of therapies and diagnostic and genetic testing alternatives. Comparison under uncertainty of incremental cost, effects and cost effectiveness across more than two strategies is conceptually and practically very different from that for two strategies, where all evidence can be summarized in a single bivariate distribution on the incremental cost-effectiveness plane. Alternative methods for comparing multiple strategies in HTA have been developed in (i) presenting cost and effects on the cost-disutility plane and (ii) summarizing evidence with multiple strategy cost-effectiveness acceptability (CEA) and expected net loss (ENL) curves and frontiers. However, critical questions remain for the analyst and decision maker of how these techniques can be best employed across multiple strategies to (i) inform clinical and cost inference in presenting evidence, and (ii) summarize evidence of cost effectiveness to inform societal reimbursement decisions where preferences may be risk neutral or somewhat risk averse under the Arrow-Lind theorem.We critically consider how evidence across multiple strategies can be best presented and summarized to inform inference and societal reimbursement decisions, given currently available methods. In the process, we make a number of important original findings.First, in presenting evidence for multiple strategies, the joint distribution of costs and effects on the cost-disutility plane with associated flexible comparators varying across replicates for cost and effect axes ensure full cost and effect inference. Such inference is usually confounded on the cost-effectiveness plane with comparison relative to a fixed origin and axes.Second, in summarizing evidence for risk-neutral societal decision making, ENL curves and frontiers are shown to have advantages over the CEA frontier in directly presenting differences in expected net benefit (ENB). The CEA frontier, while identifying strategies that maximize ENB, only presents their probability of maximizing net benefit (NB) and, hence, fails to explain why strategies maximize ENB at any given threshold value.Third, in summarizing evidence for somewhat risk-averse societal decision making, trade-offs between the strategy maximizing ENB and other potentially optimal strategies with higher probability of maximizing NB should be presented over discrete threshold values where they arise. However, the probabilities informing these trade-offs and associated discrete threshold value regions should be derived from bilateral CEA curves to prevent confounding by other strategies inherent in multiple strategy CEA curves.Based on these findings, a series of recommendations are made for best presenting and summarizing cost-effectiveness evidence for reimbursement decisions when comparing multiple strategies, which are contrasted with advice for comparing two strategies. Implications for joint research and reimbursement decisions are also discussed.