Briggs Andrew H, Doyle-Connolly Alexis, Schneider John, Podkonjak Tanja, Taylor Helen, Roffe Emma, Low Eric, Davis Sarah, Kaiser Martin, Hatswell Anthony J, Rabin Neil
London School of Hygiene and Tropical Medicine, London, UK; Avalon Health Economics, Morristown, NJ, USA.
Avalon Health Economics, Morristown, NJ, USA.
Value Health. 2025 Jan;28(1):72-80. doi: 10.1016/j.jval.2024.08.012. Epub 2024 Oct 29.
The use of cost-effectiveness methods to support policy decisions has become well established, but difficulties can arise when evaluating a new treatment that is indicated to be used in combination with an established backbone treatment. If the latter has been priced close to the decision maker's willingness-to-pay threshold, this may mean that there is no headroom for the new treatment to demonstrate value, at any price, even if the combination is clinically effective. Without a mechanism for attributing value to component treatments within a combination therapy, the health system risks generating negative funding decisions for combinations of proven clinical benefit to patients. The aim of this work was to define a value attribution methodology, which could be used to allocate value between the components of any combination treatment.
The framework is grounded in the standard decision rules of cost-effectiveness analysis and provides solutions according to key features of the problem: perfect/imperfect information about component treatment monotherapy effects and balanced/unbalanced market power between their manufacturers.
The share of incremental value varies depending on whether there is perfect/imperfect information and balance/imbalance of market power, with some scenarios requiring the manufacturers to negotiate a share of the incremental value within a range defined by the framework.
It is possible to define a framework that is independent of price and focuses on benefits expressed as quality-adjusted life-year gains (and/or quality-adjusted life-year equivalents for cost savings), a standard metric used by many health technology assessment agencies to evaluate novel treatments.
运用成本效益方法来支持政策决策已得到广泛认可，但在评估一种新疗法时可能会出现困难，这种新疗法被指定与一种既定的基础疗法联合使用。如果后者的定价接近决策者的支付意愿阈值，这可能意味着新疗法无论定价多少都没有空间来证明其价值，即使联合疗法在临床上是有效的。如果没有一种机制来为联合疗法中的各个组成疗法赋予价值，卫生系统可能会对已证明对患者有临床益处的联合疗法做出负面的资金决策。这项工作的目的是定义一种价值归属方法，可用于在任何联合治疗的各个组成部分之间分配价值。
该框架基于成本效益分析的标准决策规则，并根据问题的关键特征提供解决方案：关于组成疗法单一疗法效果的完美/不完美信息，以及其制造商之间市场力量的平衡/不平衡。
增量价值的份额因信息是否完美以及市场力量是否平衡而异，在某些情况下，需要制造商在框架定义的范围内协商增量价值的份额。
有可能定义一个独立于价格的框架，该框架侧重于以质量调整生命年增益（和/或成本节约的质量调整生命年等效值）表示的效益，这是许多卫生技术评估机构用于评估新疗法的标准指标。
