Response to the Letter to the Editor

E. Broughton
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Abstract

I greatly appreciate the comments offered by Drs Krug and Crott in ‘The “How” of Cost-Effectiveness Analysis is not so straightforward’. While I agree with several points made in their response to my original article, there are others I disagree with strongly. There are several potential sources of bias in any economic analysis, including cost-effectiveness analyses (CEA) of a care pathway. However, comparing implementation of a care pathway to an existing situation where a care pathway is not always used, and therefore current practice is ‘inappropriate’, is not one of them. Clearly, care pathways should be evidence based and it should be widely accepted that they lead to the best patient results given current knowledge. What I proposed in my original article was analysis of the incremental costs and effects of increasing the use of a care pathway from its current level in a given setting, which could be as low as no use, to as high a level of use as possible with the implementation being tested. I cannot see where the issue of bias comes into play with this basic comparison. However, I agree that generalizability of the results is often an issue and that researchers should bear this in mind when designing studies and communicating their results. Krug and Crott make a valid point on the importance of considering perspectives. Researchers should be completely explicit when stating the perspective used in the analysis, explain the reasoning behind their choices, and what would happen to the results if other costs and consequences considering different perspectives were included. I also agree with the authors on the sensitivity of results to the time horizon used for the analysis. What bears closer examination is what resources are needed to keep compliance with care pathways at a maximum level for the length of time chosen for the time horizon or, alternatively, what will happen to compliance with the care pathway beyond initial implementation if no resources are dedicated to maintaining it. Quite often these data are not available. Non-medical costs are clearly difficult to measure in almost all circumstances. Whether or not the researcher includes these costs is entirely dependent on the specific research question, which in turn depends of the needs of those commissioning the study. Given the choice, and assuming research resources are available, the societal perspective including patient and family costs should be included. The issue of what to use for an effectiveness measure in many cases involves a trade-off as presented in the original paper. To label my original discussion of this a ‘flaw’ is unwarranted. Using more accurate intermediate or process measures in the absence of good epidemiological data linking that outcome to something more tangible, such as life-years saved, means that the researcher is trading off the ability to compare results with future studies of effectiveness for a defensibly accurate result. However, if future research does accurately link process measures to tangible outcomes, the original cost-effectiveness study could be re-analyzed to yield results with a common denominator using the newly available data. Beginning with a ‘thorough and systematic’ review of relevant literature for these or any studies is de riguer and should require no reiteration. The purpose of my original paper was, as clearly stated, to provide elementary information for CEAs for care pathways in the hope that it would lead to greater appreciation and support for this kind of research. It was not written to delve deeply into the details of performing such a study. There are several reference books that serve this purpose admirably in the expansive ways necessary for such detail. However misguided several of the criticisms given by Krug and Crott were, I appreciate the attention it brings to this very important topic and hope that it contributes to making economic analyses of care pathway implementation more numerous, more rigorous and more widely used.
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我非常感谢Krug和Crott博士在《成本效益分析的“方法”不是那么直截了当》一文中提出的意见。虽然我同意他们对我最初文章的回应中的一些观点,但也有一些我强烈反对的观点。在任何经济分析中都有几个潜在的偏倚来源,包括护理途径的成本效益分析(CEA)。然而,将护理途径的实施与并不总是使用护理途径的现有情况进行比较,因此目前的做法是“不适当的”,这不是其中之一。显然,护理路径应该以证据为基础,并且应该被广泛接受,即根据目前的知识,它们会导致最佳的患者结果。我在最初的文章中提出的建议是分析在给定环境中增加护理途径的使用的增量成本和效果,从目前的水平(可能低到没有使用)增加到尽可能高的使用水平,并进行实施测试。我看不出偏见的问题在这个基本的比较中起作用。然而,我同意结果的普遍性经常是一个问题,研究人员在设计研究和交流结果时应该牢记这一点。Krug和Crott在考虑观点的重要性上提出了一个有效的观点。研究人员在陈述分析中使用的视角时应该完全明确,解释他们选择背后的原因,以及如果考虑到不同视角的其他成本和后果,结果会发生什么。我也同意作者关于结果对用于分析的时间范围的敏感性。需要仔细研究的是,在选定的时间范围内,需要哪些资源来保持对护理途径的最大程度的遵守,或者,如果没有专门的资源来维持,那么在最初实施之后,对护理途径的遵守将会发生什么情况。通常这些数据是不可用的。在几乎所有情况下,非医疗成本显然都难以衡量。研究人员是否包括这些费用完全取决于具体的研究问题,这反过来又取决于委托研究的人的需求。如果有选择,并且假设研究资源是可用的,那么应从包括患者和家庭成本在内的社会角度考虑。在许多情况下,使用什么作为有效性度量的问题涉及到原始论文中提出的权衡。将我最初的讨论贴上“缺陷”的标签是没有根据的。在缺乏将结果与更具体的东西(如节省的寿命)联系起来的良好流行病学数据的情况下,使用更准确的中间或过程测量,意味着研究人员正在权衡将结果与未来有效性研究进行比较的能力,以获得可辩护的准确结果。然而,如果未来的研究确实准确地将过程措施与有形结果联系起来,那么可以重新分析原始的成本效益研究,以使用新获得的数据产生具有公分母的结果。首先对这些或任何研究的相关文献进行“彻底和系统”的回顾是必要的,不需要重复。正如明确指出的那样,我最初论文的目的是为护理途径的cea提供基本信息,希望它能导致对这类研究的更多赞赏和支持。它并不是为了深入研究进行这样一项研究的细节而写的。有几本参考书很好地服务于这一目的,以必要的广泛方式提供了这样的细节。无论Krug和Crott给出的一些批评有多么误导,我都很欣赏它给这个非常重要的话题带来的关注,并希望它有助于对护理途径实施的经济分析进行更多、更严格和更广泛的应用。
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