Clinical Pharmacology, Modeling & Simulation, GlaxoSmithKline, PO Box 13398, 5 Moore Drive, Research Triangle Park, NC 27709-3398, USA.
Expert Opin Drug Saf. 2010 Mar;9(2):289-300. doi: 10.1517/14740330903499265.
Measurement of the tradeoff between efficacy and safety is rarely done in a quantitative fashion. The use of a clinical utility index (CUI) has been proposed as a tool to aid in this assessment. The methodology from multi-attribute decision analysis is in its early stage in drug development and can be used to understand the therapeutic index and the value relative to the competitive landscape.
Different examples and applications of the use of CUI are reviewed and key steps involved in the development described. These include: i) characterization of the exposure-response of efficacy and safety end points; ii) definition of clinically meaningful parameters; iii) selection and weighting of important attributes and iv) sensitivity analysis and measurement of uncertainty.
An understanding of the value and limitations of CUI in drug development.
The use of a CUI for quantitative assessment of benefit/risk is most useful when there are multiple attributes involved in a decision to better understand the relevance of each attribute and when differentiation from competitors is critical to the success of a compound. Although development of CUI may be time- and resource-consuming, it allows clear and transparent decision making.
很少以定量方式来衡量疗效和安全性之间的权衡取舍。已经提出使用临床实用指数（CUI）作为辅助评估的工具。多属性决策分析方法在药物开发中还处于早期阶段，可以用于了解治疗指数和相对于竞争格局的价值。
综述了 CUI 的不同示例和应用，并描述了其开发过程中的关键步骤。这些步骤包括：i）对疗效和安全性终点的暴露-反应进行特征描述；ii）定义有临床意义的参数；iii）选择和加权重要属性；iv）敏感性分析和不确定性测量。
了解 CUI 在药物开发中的价值和局限性。
当决策中涉及多个属性，以便更好地了解每个属性的相关性，并且区分竞争对手对于化合物的成功至关重要时，使用 CUI 进行收益/风险的定量评估最为有用。虽然开发 CUI 可能需要时间和资源，但它允许做出明确和透明的决策。
