Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, Massachusetts.
Evidence for Access, Public Affairs and Access, Genentech, San Francisco, California.
JAMA Netw Open. 2024 Oct 1;7(10):e2442353. doi: 10.1001/jamanetworkopen.2024.42353.
The arrival of new medications for Alzheimer disease (AD) has prompted efforts to measure their value using conventional cost-effectiveness analyses; however, these analyses focus on how much health improvement new medications generate per dollar spent. As AD disproportionately affects older adults, women, racial and ethnic minority individuals, and individuals with lower socioeconomic and educational levels, it is critical to also examine the health equity outcomes of treatment.
To estimate the health equity impact of a hypothetical disease-modifying treatment for early AD in the US and to examine targeted policies to mitigate health care disparities.
DESIGN, SETTING, AND PARTICIPANTS: This economic evaluation, which used a distributional cost-effectiveness analysis, was conducted from June 16, 2022, to January 11, 2024. The study included subgroups defined by race and ethnicity and by social vulnerability quintiles in the US.
A hypothetical disease-modifying treatment compared with best supportive care.
The main outcomes were population-level quality-adjusted life-years (QALYs), lifetime costs, and net health benefits. The social welfare impact and change in health inequality were estimated using the Atkinson index.
The distributional cost-effectiveness analysis simulated 316 037 100 individuals from the US population, including 25 subgroups defined by 5 categories of race and ethnicity and population quintiles of social vulnerability, with the fifth quintile representing the most socially vulnerable group. At an opportunity cost benchmark of $150 000 per QALY, treatment was associated with improved population health, adding 28 197 QALYs per year to the US population. Accounting for health inequality preferences (using an aversion level of 11, based on an Atkinson inequality aversion parameter that can range from 0 to infinity, with higher values assigning greater weight to health gains that accrue to the population with the lowest lifetime quality-adjusted life expectancy), treatment was associated with a 0.009% reduction in existing population health inequalities annually. Scenario analyses examining earlier and expanded treatment access suggested a population health improvement of up to 221 358 QALYs.
The findings of this economic evaluation suggest that treatment for AD could improve population health and health equity. Policies to enable earlier diagnosis and treatment initiation, as well as expanded access to treatment, may further improve treatment and health equity impacts.
新的阿尔茨海默病（AD）药物的出现促使人们努力使用传统的成本效益分析来衡量它们的价值；然而，这些分析侧重于新药物每花费一美元能产生多少健康改善。由于 AD 不成比例地影响老年人、女性、种族和少数民族个人以及社会经济和教育水平较低的个人，因此，评估治疗的健康公平结果至关重要。
估计在美国，一种假设的用于早期 AD 的疾病修正治疗的健康公平影响，并研究针对减轻医疗保健差距的有针对性的政策。
设计、设置和参与者：这项经济评估使用了分布成本效益分析，于 2022 年 6 月 16 日至 2024 年 1 月 11 日进行。该研究包括美国按种族和族裔以及社会脆弱性五分位数定义的亚组。
与最佳支持性治疗相比，一种假设的疾病修正治疗。
主要结果是人群水平的质量调整生命年（QALYs）、终生成本和净健康收益。使用阿特金森指数估计社会福利影响和健康不平等的变化。
分布成本效益分析模拟了来自美国人口的 316037100 个人，包括 25 个亚组，分为种族和族裔的 5 个类别和社会脆弱性的人口五分位数，第五个五分位数代表最脆弱的群体。在机会成本基准为 150000 美元/QALY 的情况下，治疗与改善人群健康相关，每年为美国人口增加 28197 个 QALYs。考虑到健康不平等偏好（使用基于阿特金森不平等厌恶参数的 11 作为厌恶水平，该参数的范围可以从 0 到无穷大，较高的值对分配给预期寿命最低的人群的健康收益赋予更大的权重），治疗与每年人口健康不平等减少 0.009%相关。检查更早和扩大治疗机会的情景分析表明，人口健康改善高达 221358QALYs。
这项经济评估的结果表明，AD 的治疗可能会改善人群健康和健康公平。使早期诊断和治疗开始以及扩大治疗机会的政策可能会进一步改善治疗和健康公平的影响。
