Rogowski Wolf, Payne Katherine, Schnell-Inderst Petra, Manca Andrea, Rochau Ursula, Jahn Beate, Alagoz Oguzhan, Leidl Reiner, Siebert Uwe
Helmholtz Zentrum München, Institute of Health Economics and Health Care Management, Ingolstädter Landstr. 1, 85764, Neuherberg, Germany,
Pharmacoeconomics. 2015 Jan;33(1):49-59. doi: 10.1007/s40273-014-0211-5.
This study assesses if, and how, existing methods for economic evaluation are applicable to the evaluation of personalized medicine (PM) and, if not, where extension to methods may be required.
A structured workshop was held with a predefined group of experts (n = 47), and was run using a modified nominal group technique. Workshop findings were recorded using extensive note taking, and summarized using thematic data analysis. The workshop was complemented by structured literature searches.
The key finding emerging from the workshop, using an economic perspective, was that two distinct, but linked, interpretations of the concept of PM exist (personalization by 'physiology' or 'preferences'). These interpretations involve specific challenges for the design and conduct of economic evaluations. Existing evaluative (extra-welfarist) frameworks were generally considered appropriate for evaluating PM. When 'personalization' is viewed as using physiological biomarkers, challenges include representing complex care pathways; representing spillover effects; meeting data requirements such as evidence on heterogeneity; and choosing appropriate time horizons for the value of further research in uncertainty analysis. When viewed as tailoring medicine to patient preferences, further work is needed regarding revealed preferences, e.g. treatment (non)adherence; stated preferences, e.g. risk interpretation and attitude; consideration of heterogeneity in preferences; and the appropriate framework (welfarism vs. extra-welfarism) to incorporate non-health benefits.
Ideally, economic evaluations should take account of both interpretations of PM and consider physiology and preferences. It is important for decision makers to be cognizant of the issues involved with the economic evaluation of PM to appropriately interpret the evidence and target future research funding.
本研究评估现有经济评估方法是否适用于个性化医疗（PM）的评估，若不适用，哪些方法可能需要扩展。
与一组预先确定的专家（n = 47）举办了一次结构化研讨会，采用改良的名义群体技术进行。通过大量记录笔记来记录研讨会结果，并使用主题数据分析进行总结。通过结构化文献检索对研讨会进行补充。
从研讨会中得出的关键发现，从经济学角度来看，存在对PM概念的两种不同但相关的解释（基于“生理学”或“偏好”的个性化）。这些解释给经济评估的设计和实施带来了特定挑战。现有的评估（超福利主义）框架通常被认为适用于评估PM。当“个性化”被视为使用生理生物标志物时，挑战包括呈现复杂的护理路径；呈现溢出效应；满足数据要求，如关于异质性的证据；以及在不确定性分析中为进一步研究的价值选择合适的时间范围。当被视为根据患者偏好定制医疗时，在显示偏好方面，例如治疗（不）依从性；陈述偏好方面，例如风险解读和态度；偏好异质性的考虑；以及纳入非健康益处的合适框架（福利主义与超福利主义）等方面还需要进一步开展工作。
理想情况下，经济评估应考虑PM的两种解释，并兼顾生理学和偏好。决策者认识到PM经济评估所涉及的问题对于正确解读证据和确定未来研究资金的投向很重要。
