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临床研究中的复合结局测量:是幻想战胜了现实吗?

Composite outcome measurement in clinical research: the triumph of illusion over reality?

作者信息

McKenna Stephen P, Heaney Alice

机构信息

Galen Research Ltd., Manchester, UK.

School of Health Sciences, University of Manchester, Manchester, UK.

出版信息

J Med Econ. 2020 Oct;23(10):1196-1204. doi: 10.1080/13696998.2020.1797755. Epub 2020 Jul 29.

Abstract

Composite measures that combine different types of indicators are widely used in medical research; to evaluate health systems, as outcomes in clinical trials and patient-reported outcome measurement. The potential advantages of such indices are clear. They are used to summarise complex data and to overcome the problem of evaluating new interventions when the most important outcome is rare or likely to occur far in the future. However, many scientists question the value of composite measures, primarily due to inadequate development methodology, lack of transparency or the likelihood of producing misleading results. It is argued that the real problems with composite measurement are related to their failure to take account of measurement theory and the absence of coherent theoretical models that justify the addition of the individual indicators that are combined into the composite index. All outcome measures must be unidimensional if they are to provide meaningful data. They should also have dimensional homogeneity. Ideally, a specification equation should be developed that can predict accurately how organisations or individuals will score on an index, based on their scores on the individual indicators that make up the measure. The article concludes that composite measures should not be used as they fail to apply measurement theory and, consequently, produce invalid and misleading scores.

摘要

综合不同类型指标的复合指标在医学研究中被广泛使用;用于评估卫生系统、作为临床试验的结果以及患者报告的结局测量。此类指标的潜在优势显而易见。它们用于汇总复杂数据,并克服在最重要的结局罕见或可能在遥远未来发生时评估新干预措施的问题。然而,许多科学家质疑复合指标的价值,主要是因为开发方法不完善、缺乏透明度或可能产生误导性结果。有人认为,复合测量的真正问题与其未能考虑测量理论以及缺乏连贯的理论模型有关,这些理论模型可为纳入复合指标的各个指标的相加提供依据。所有结局指标若要提供有意义的数据,必须是单维的。它们还应具有维度同质性。理想情况下,应开发一个规格方程,该方程可根据构成该测量的各个指标的得分准确预测组织或个人在一个指标上的得分。文章得出结论,复合指标不应被使用,因为它们未能应用测量理论,因此会产生无效和误导性的分数。

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