Tervonen Tommi, Naci Huseyin, van Valkenhoef Gert, Ades Anthony E, Angelis Aris, Hillege Hans L, Postmus Douwe
Econometric Institute, Erasmus School of Economics, Erasmus University Rotterdam, The Netherlands (TT)
LSE Health, London School of Economics and Political Science, London, UK (HN, AA)
Med Decis Making. 2015 Oct;35(7):859-71. doi: 10.1177/0272989X15587005. Epub 2015 May 18.
Decision makers in different health care settings need to weigh the benefits and harms of alternative treatment strategies. Such health care decisions include marketing authorization by regulatory agencies, practice guideline formulation by clinical groups, and treatment selection by prescribers and patients in clinical practice. Multiple criteria decision analysis (MCDA) is a family of formal methods that help make explicit the tradeoffs that decision makers accept between the benefit and risk outcomes of different treatment options. Despite the recent interest in MCDA, certain methodological aspects are poorly understood. This paper presents 7 guidelines for applying MCDA in benefit-risk assessment and illustrates their use in the selection of a statin drug for the primary prevention of cardiovascular disease. We provide guidance on the key methodological issues of how to define the decision problem, how to select a set of nonoverlapping evaluation criteria, how to synthesize and summarize the evidence, how to translate relative measures to absolute ones that permit comparisons between the criteria, how to define suitable scale ranges, how to elicit partial preference information from the decision makers, and how to incorporate uncertainty in the analysis. Our example on statins indicates that fluvastatin is likely to be the most preferred drug by our decision maker and that this result is insensitive to the amount of preference information incorporated in the analysis.
不同医疗环境中的决策者需要权衡不同治疗策略的利弊。此类医疗决策包括监管机构的上市许可、临床团体制定的实践指南,以及临床实践中开处方者和患者的治疗选择。多标准决策分析(MCDA)是一系列正式方法,有助于明确决策者在不同治疗方案的获益和风险结果之间所接受的权衡。尽管最近人们对MCDA很感兴趣,但某些方法学方面仍未得到充分理解。本文提出了在获益-风险评估中应用MCDA的7条指南,并说明了它们在选择用于心血管疾病一级预防的他汀类药物中的应用。我们就如何定义决策问题、如何选择一组不重叠的评估标准、如何综合和总结证据、如何将相对指标转化为可在不同标准间进行比较的绝对指标、如何定义合适的量表范围、如何从决策者那里获取部分偏好信息,以及如何在分析中纳入不确定性等关键方法学问题提供指导。我们关于他汀类药物的示例表明,氟伐他汀可能是我们的决策者最青睐的药物,而且这一结果对分析中纳入的偏好信息量不敏感。