Canadian Centre for Applied Research in Cancer Control, Cancer Control Research, BC Cancer Research Centre, 675 W. 10th Ave, Vancouver, BC, V5Z 1L3, Canada.
School of Population and Public Health, University of British Columbia, Vancouver, BC, Canada.
Pharmacoeconomics. 2022 Oct;40(10):931-941. doi: 10.1007/s40273-022-01176-0. Epub 2022 Jul 27.
Precision medicine highlights the importance of exploring heterogeneity in the effectiveness and costs of interventions. Our objective was to identify and compare frameworks for valuing heterogeneity-informed decisions, and consider their strengths and weaknesses for application to precision medicine.
We conducted a scoping review to identify papers that proposed an analytical framework to place a value, in terms of costs and health benefits, on using heterogeneity to inform treatment selection. The search included English-language papers indexed in MEDLINE, Embase or EconLit, and a manual review of references and citations. We compared the frameworks qualitatively considering: the purpose and setting of the analysis; the types of precision medicine interventions where the framework could be applied; and the framework's ability to address the methodological challenges of evaluating precision medicine.
Four analytical frameworks were identified: value of stratification, value of heterogeneity, expected value of individualised care and loss with respect to efficient diffusion. Each framework is suited to slightly different settings and research questions. All focus on maximising net benefit, and quantify the opportunity cost of ignoring heterogeneity by comparing individualised or stratified decisions to a means-based population-wide decision. Where the frameworks differ is in their approaches to uncertainty, and in the additional metrics they consider.
Identifying and utilising heterogeneity is at the core of precision medicine, and the ability to quantify the value of heterogeneity-informed decisions is critical. Using an analytical framework to value heterogeneity will help provide evidence to inform investment in precision medicine interventions, appropriately capturing the value of targeted health interventions.
精准医学强调了探索干预措施效果和成本异质性的重要性。我们的目标是识别和比较用于评估异质信息决策的框架，并考虑它们在应用于精准医学方面的优缺点。
我们进行了范围综述，以确定提出分析框架的论文，该框架根据使用异质信息来指导治疗选择的成本和健康效益来评估价值。该搜索包括在 MEDLINE、Embase 或 EconLit 中索引的英文论文，并对参考文献和引文进行了手动审查。我们从以下几个方面对框架进行了定性比较：分析的目的和背景；该框架可应用于哪种类型的精准医学干预；以及该框架解决评估精准医学的方法学挑战的能力。
确定了四个分析框架：分层价值、异质价值、个体化护理的预期价值和有效扩散的损失。每个框架都适用于略有不同的设置和研究问题。所有框架都侧重于最大化净收益，并通过将个体化或分层决策与基于均值的人群决策进行比较，量化忽略异质性的机会成本。这些框架的不同之处在于其对不确定性的处理方式，以及它们考虑的其他指标。
识别和利用异质性是精准医学的核心，量化异质信息决策的价值至关重要。使用分析框架来评估异质性将有助于提供证据，为精准医学干预措施的投资提供信息，合理捕捉靶向健康干预措施的价值。
