Keim-Malpass Jessica, Malpass H Charles
University of Virginia School of Nursing, Charlottesville, VA, USA.
Department of Pediatrics, University of Virginia School of Medicine, Charlottesville, VA, USA.
Clinicoecon Outcomes Res. 2022 Jun 17;14:427-437. doi: 10.2147/CEOR.S362530. eCollection 2022.
Asthma is a disease with tremendous phenotypic heterogeneity, and the patients who are most severely impacted by the disease are high utilizers of the United States healthcare system. In the past decade, there has been many advances in asthma therapy for those with severe disease, including the use of a procedure called bronchial thermoplasty (BT) and the use of biologic therapy for certain phenotypes, but questions remain regarding the long-term durability and cost effectiveness of these therapies. The purpose of this analysis was (1) to assess the cost utility of BT relative to usual care (base case) and (2) to assess the cost utility of BT relative to usual care plus biologic therapy (omalizumab) (scenario analysis) based on updated 10-year clinical trial outcomes.
A Markov cohort model was developed and used to estimate the cost utility of BT to estimate the costs and quality-of-life impact of BT versus the comparisons over a 10-year time frame using a limited societal perspective, which included both direct health utilization costs and indirect costs associated with missed days of work, among those with severe persistent asthma.
In the base case and the scenario analysis, BT was the dominant treatment strategy compared to usual care alone and usual care plus biologic therapy. The net monetary benefit for BT was $483,555.49 over a 10-year time horizon.
Cost-utility models are central to policy decisions dictating coverage, and can be extended to inform the patient and provider, during clinical decision-making, of the relative trade-offs of therapy, assessing long-term clinical and cost outcomes. Phenotypic classification of severe asthma is central to patient management and should also be integrated into economic analysis frameworks, particularly as new biologic agents are developed that are specific to a phenotype. Despite a larger upfront cost of BT therapy, there is a durable clinical and economic benefit over time for those with severe asthma.
哮喘是一种具有巨大表型异质性的疾病，受该疾病影响最严重的患者是美国医疗保健系统的高使用者。在过去十年中，针对重症患者的哮喘治疗取得了许多进展，包括使用一种名为支气管热成形术（BT）的程序以及针对某些表型使用生物疗法，但这些疗法的长期疗效和成本效益仍存在疑问。本分析的目的是：（1）评估BT相对于常规治疗（基础病例）的成本效用；（2）基于更新的10年临床试验结果，评估BT相对于常规治疗加生物疗法（奥马珠单抗）的成本效用（情景分析）。
开发了一个马尔可夫队列模型，并用于估计BT的成本效用，以使用有限的社会视角估计BT与对照在10年时间范围内的成本和生活质量影响，其中包括直接医疗利用成本以及与旷工相关的间接成本，研究对象为重度持续性哮喘患者。
在基础病例和情景分析中，与单独的常规治疗以及常规治疗加生物疗法相比，BT是主要的治疗策略。在10年时间范围内，BT的净货币效益为483,555.49美元。
成本效用模型对于决定保险覆盖范围的政策决策至关重要，并且可以扩展以在临床决策过程中告知患者和医疗服务提供者治疗的相对权衡，评估长期临床和成本结果。重症哮喘的表型分类对于患者管理至关重要，也应纳入经济分析框架，特别是随着针对特定表型的新型生物制剂的开发。尽管BT治疗的前期成本较高，但对于重度哮喘患者来说，随着时间的推移会有持久的临床和经济效益。
