Cost-utility and cost-benefit analysis of TAVR availability in the US severe symptomatic aortic stenosis patient population

We evaluated the availability of transcatheter aortic valve replacement (TAVR) to determine its value across all severe symptomatic aortic stenosis (SSAS) patients, especially those untreated because of concerns regarding invasive surgical AVR (SAVR) and its impact on active aging. We performed payer perspective cost-utility analysis (CUA) and societal perspective cost-benefit analysis (CBA). The CBA’s benefit measure is active time: salaried labor, unpaid work, and active leisure. The study population is a cohort of US elderly SSAS patients. We compared a “TAVR available” scenario in which SSAS patients distribute themselves across TAVR, SAVR, and medical management (MM); and a “TAVR not available” scenario with only SAVR and MM. We structured each scenario with a decision-tree model of SSAS patient treatment allocation. We measured the association between health and active time in the US Health and Retirement Study and used this association to impute active time to SSAS patients given their health. The incremental cost-effectiveness ratio (ICER) and rate of return (RoR) of TAVR availability were $8,533 and 395%, respectively. CUA net monetary benefits (NMB) were $212,199 per patient and $43.4 billion population-wide. CBA NMB were $50,530 per patient and $10.3 billion population-wide. Among study limitations were scarcity of evidence regarding key parameters and the lack of long-term survival, health utility, and treatment cost data. Our analysis did not account for TAVR durability, retreatments, and valve-in-valve treatments. Across risk-, age-, and treatment-eligibility groups, TAVR is the economically optimal treatment choice. It represents strong value-for-money per patient and population-wide. The vast majority of TAVR value involves raising treatment uptake among the untreated. Aortic stenosis (AS) is a common and lethal heart disease. Surgical treatment has long been available, but its invasiveness limits uptake. More recently, transcatheter aortic valve replacement (TAVR) has emerged as a treatment alternative. Its minimal invasiveness has significantly increased treatment rates, but economic evaluations omit this benefit, risking undervaluation. We evaluated TAVR in elderly US severe symptomatic AS patients, using payer perspective cost-utility analysis (CUA) and societal perspective cost-benefit analysis (CBA). Both CUA and CBA incorporated TAVR’s impact on treatment rates. Given patient preferences for treatment options promoting active aging, our CBA used the value of active time as a benefit measure. We found that CUA/CBA net monetary benefits are $212,199/$50,530 per patient. Across risk-, age-, and treatment-eligibility groups, TAVR is the economically optimal treatment choice over surgery and medical management. It represents strong value-for-money per patient and population-wide. Increased treatment uptake accounts for the vast share of TAVR's value.

本研究评估了经导管主动脉瓣置换术（transcatheter aortic valve replacement, TAVR）的可及性，以明确其在所有症状性重度主动脉瓣狭窄（severe symptomatic aortic stenosis, SSAS）患者中的临床价值，尤其是那些因担忧侵入性外科主动脉瓣置换术（surgical AVR, SAVR）及其对积极老龄化的影响而未接受治疗的患者。本研究采用支付方视角开展成本效用分析（cost-utility analysis, CUA），并采用社会视角开展成本效益分析（cost-benefit analysis, CBA）。成本效益分析的效益衡量指标为活动时长：包括带薪劳动、无偿工作与休闲活动时长。本研究的研究人群为美国老年症状性重度主动脉瓣狭窄患者队列。本研究设置两种场景进行对比：其一为“TAVR可及”场景，此时症状性重度主动脉瓣狭窄患者可选择经导管主动脉瓣置换术、外科主动脉瓣置换术或药物治疗（medical management, MM）；其二为“TAVR不可及”场景，此时患者仅可选择外科主动脉瓣置换术或药物治疗。本研究通过症状性重度主动脉瓣狭窄患者治疗分配决策树模型构建上述两种场景。本研究基于美国健康与退休研究（US Health and Retirement Study）分析了健康状况与活动时长之间的关联，并据此根据症状性重度主动脉瓣狭窄患者的健康状况推算其活动时长。经导管主动脉瓣置换术可及性对应的增量成本效用比（incremental cost-effectiveness ratio, ICER）与回报率（rate of return, RoR）分别为8533美元与395%。成本效用分析的净货币效益（net monetary benefits, NMB）为每名患者212199美元，全人群总计434亿美元；成本效益分析的净货币效益为每名患者50530美元，全人群总计103亿美元。本研究存在若干局限性：关键参数相关证据较为匮乏，且缺乏长期生存、健康效用与治疗成本相关数据。本研究未考虑经导管主动脉瓣置换术的耐久性、再治疗需求以及瓣中瓣治疗（valve-in-valve treatments）相关因素。在不同风险分层、年龄分组与治疗资格分组中，经导管主动脉瓣置换术均为经济学最优治疗方案。无论从单患者视角还是全人群视角，经导管主动脉瓣置换术均具备极佳的成本效益比。经导管主动脉瓣置换术的绝大部分价值来源于提升未接受治疗患者的治疗普及率。
主动脉瓣狭窄（aortic stenosis, AS）是一种常见且致命的心脏疾病。外科治疗长期以来均为可选方案，但其侵入性特征限制了治疗普及率。近年来，经导管主动脉瓣置换术（TAVR）作为替代治疗方案应运而生。其微创性显著提升了治疗普及率，但现有经济学评价往往忽略这一效益，存在低估其价值的风险。本研究针对美国老年症状性重度主动脉瓣狭窄患者，采用支付方视角成本效用分析与社会视角成本效益分析评估经导管主动脉瓣置换术的价值。两类分析均纳入了经导管主动脉瓣置换术对治疗普及率的影响。考虑到患者偏好可促进积极老龄化的治疗方案，本研究的成本效益分析以活动时长价值作为效益衡量指标。本研究结果显示，成本效用分析与成本效益分析的净货币效益分别为每名患者212199美元与50530美元。在不同风险分层、年龄分组与治疗资格分组中，经导管主动脉瓣置换术均优于外科治疗与药物治疗，为经济学最优方案。无论单患者视角还是全人群视角，经导管主动脉瓣置换术均具备极佳的成本效益比。治疗普及率的提升是经导管主动脉瓣置换术价值的主要来源。