Quintiles Professor of Pharmaceutical Development and Regulatory Innovation, School of Pharmacy, Price School of Public Policy, Leonard Schaeffer Center for Health Policy and Economics, University of Southern California, Los Angeles, CA, USA; National Bureau of Economic Research, Cambridge, MA, USA.
University Professor and Provost Emeritus, University of Rochester, Rochester, NY, USA.
Value Health. 2021 Feb;24(2):244-249. doi: 10.1016/j.jval.2020.10.003. Epub 2021 Jan 12.
Cost-effectiveness analysis (CEA) embeds an assumption at odds with most economic analysis-that of constant returns to health in the creation of happiness (utility). We aim to reconcile it with the bulk of economic theory.
We generalize the traditional CEA approach, allow diminishing returns to health, and align CEA with the rest of the health economics literature.
This simple change has far-reaching implications for the practice of CEA. First, optimal cost-effectiveness thresholds should systematically rise for more severe diseases and fall for milder ones. We provide formulae for estimating how these thresholds vary with health-related quality of life (QoL) in the sick state. Practitioners can also use our approach to account for treatment outcome uncertainty. Holding average benefits fixed, risk-averse consumers value interventions more when they reduce outcome uncertainty ('insurance value') and/or when they provide a chance at positively skewed outcomes ('value of hope'). Finally, we provide a coherent way to combine improvements in QoL and life expectancy (LE) when people have diminishing returns to QoL.
This new approach obviates the need for increasingly prevalent and ad hoc exceptions to CEA for end-of-life care, rare disease, and very severe disease (eg, cancer). Our methods also show that the value of improving QoL for disabled people is greater than for comparable non-disabled people, thus resolving an ongoing and mathematically legitimate objection to CEA raised by advocates for disabled people. Our Generalized Risk-Adjusted Cost-Effectiveness (GRACE) approach helps align HTA practice with realistic preferences for health and risk.
成本效益分析(CEA)嵌入了一个与大多数经济分析假设相矛盾的假设,即健康创造幸福(效用)的收益是不变的。我们旨在将其与经济理论的大部分内容相协调。
我们推广了传统的 CEA 方法,允许健康回报递减,并使 CEA 与卫生经济学文献的其他部分保持一致。
这一简单的变化对 CEA 的实践产生了深远的影响。首先,对于更严重的疾病,最优成本效益阈值应该系统地上升,而对于较轻的疾病则应该下降。我们提供了用于估计这些阈值如何随健康相关生活质量(QoL)在患病状态下变化的公式。从业者还可以使用我们的方法来考虑治疗结果不确定性。在固定平均收益的情况下,风险厌恶型消费者更看重减少结果不确定性的干预措施(“保险价值”)和/或提供正偏结果机会的干预措施(“希望价值”)。最后,我们提供了一种一致的方法来结合 QoL 和预期寿命(LE)的改善,当人们对 QoL 的回报递减时。
这种新方法避免了为临终关怀、罕见病和非常严重的疾病(如癌症)不断增加和特殊的 CEA 例外的需要。我们的方法还表明,改善残疾人士 QoL 的价值大于可比非残疾人士,从而解决了残疾人士倡导者对 CEA 提出的持续存在且在数学上合理的反对意见。我们的广义风险调整成本效益(GRACE)方法有助于使 HTA 实践与对健康和风险的现实偏好保持一致。