综合疗法的价值归属框架。

IF 4.9 2区 医学 Q1 ECONOMICS Value in Health Pub Date : 2024-10-29 DOI:10.1016/j.jval.2024.08.012
Andrew H Briggs, Alexis Doyle-Connolly, John Schneider, Tanja Podkonjak, Helen Taylor, Emma Roffe, Eric Low, Sarah Davis, Martin Kaiser, Anthony J Hatswell, Neil Rabin
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引用次数: 0

摘要

目的:使用成本效益方法来支持政策决策的做法已经非常成熟,但在评估一种新疗法时可能会遇到困难,因为这种新疗法需要与已确立的 "骨干疗法 "联合使用。如果 "骨干疗法 "的定价已接近决策者的支付意愿阈值,这可能意味着新疗法在任何价格下都没有展示价值的余地,即使这种组合在临床上是有效的。如果没有一种机制来为联合疗法中的成分治疗赋予价值,医疗系统就有可能对已证明对患者有临床益处的联合疗法做出负面的资助决定。这项工作的目的是确定一种价值归属方法,用于在任何联合疗法的各组成部分之间分配价值:方法:该框架以成本效益分析的标准决策规则为基础,并根据问题的关键特征提供解决方案:关于组合疗法单一疗法效果的完美/不完美信息,以及其制造商之间平衡/不平衡的市场力量:增量价值的份额因信息是否完全/不完全和市场力量是否平衡/不平衡而不同,有些方案要求制造商在框架定义的范围内协商增量价值的份额:可以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(QALY)收益(和/或成本节约的 QALY 当量)表示的效益,这是许多 HTA 机构用于评估新型疗法的标准指标。
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An attribution of value framework for combination treatments.

Objective: The use of cost-effectiveness methods to support policy decisions has become well established but difficulties can arise when evaluating a new treatment which 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.

Methods: 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.

Results: 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.

Conclusions: It is possible to define a framework that is independent of price and focuses on benefits expressed as Quality-Adjusted Life-Year (QALY) gains (and/or QALY equivalents for cost-savings), a standard metric used by many HTA agencies to evaluate novel treatments.

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来源期刊
Value in Health
Value in Health 医学-卫生保健
CiteScore
6.90
自引率
6.70%
发文量
3064
审稿时长
3-8 weeks
期刊介绍: Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.
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