Analysis Group, Inc., Montreal, Quebec, Canada.
Otsuka Pharmaceutical Development & Commercialization, Inc., Rockville, MD.
J Manag Care Spec Pharm. 2021 Dec;27(12):1703-1713. doi: 10.18553/jmcp.2021.27.12.1703.
Erythropoiesis-stimulating agents (ESAs) are commonly used to treat anemia due to chronic kidney disease (CKD). In addition to drug acquisition costs, the administration of ESAs can include direct and indirect costs due to the needle-based route of administration (eg, time spent by health care staff administering therapy, and patients' and caregivers' time spent receiving or assisting with therapy). However, a comprehensive assessment of the costs associated with the administration of ESAs is lacking. To estimate the excess costs associated with the needle-based administration of ESAs for the treatment of anemia due to non-dialysis-dependent (NDD) CKD in the United States in 2019 from a societal perspective. Excess costs associated with ESA administration were estimated as the sum of annual costs that could be avoided with the introduction of an oral treatment with comparable safety and efficacy to ESAs. Cost components included direct health care costs, transportation costs, and work productivity loss costs from the perspective of both patients and caregivers (as applicable). Costs were estimated based on scientific publications, governmental agencies, and the results of a recent survey of US patients and caregivers of patients with anemia and CKD. The setting of the administration (ie, at home vs in clinic), frequency of administration, and insurance type were considered. At the societal level, annual excess costs associated with ESA administration were estimated at $2.5 billion in the United States in 2019, based on an estimated 462,005 patients with anemia and NDD-CKD treated with ESAs. Overall, 94.4% ($2.4 billion) of these costs were incurred from in-clinic ESA administration. When stratifying costs by insurance type, Medicare-insured patients accounted for 79.4% ($2.0 billion) of total annual excess costs. The largest contributor to total annual excess costs was direct health care costs ($1.4 billion, 54.9%), followed by patient work productivity loss costs ($846 million, 33.9%), caregiver work productivity loss costs ($197 million, 7.9%), and transportation costs ($81 million, 3.3%). Total annual excess costs of in-clinic administration ranged from $2,572 per patient receiving monthly administration to $20,948 per patient receiving thrice-weekly administration, while the total annual excess costs of at-home administration ranged from $1,123 per patient receiving monthly administration to $2,109 per patient receiving thrice-weekly administration. At the ESA administration level (ie, for each ESA administration), total excess costs were estimated at $128 per in-clinic ESA administration and $7 per at-home ESA administration, excluding monitoring costs. The needle-based administration of ESAs in patients with NDD-CKD is associated with a substantial economic burden. The introduction of an oral treatment has the potential to result in important cost savings from a societal perspective. : This study was funded by Otsuka Pharmaceutical Development & Commercialization, Inc., and Akebia Therapeutics, Inc. The study sponsors participated in the study design, data collection, analysis, interpretation of the data, writing of the report, and in the decision to submit the manuscript for publication. Gauthier-Loiselle, Cloutier, Serra, Bungay, and Guérin are employees of Analysis Group, Inc., a consulting firm that received funding from Otsuka Pharmaceutical Development & Commercialization, Inc., for the conduct of this study. Michalopoulos was an employee of Otsuka Pharmaceutical Development & Commercialization, Inc., at the time the study was conducted. Szabo is an employee of Akebia Therapeutics, Inc.
促红细胞生成素刺激剂(ESAs)常用于治疗因慢性肾脏病(CKD)导致的贫血。除药物购置成本外,ESA 的给药还包括因给药途径为注射(例如医护人员给药所花费的时间,以及患者和护理人员接受或协助治疗所花费的时间)而产生的直接和间接成本。然而,目前缺乏对 ESA 给药相关成本的全面评估。本研究旨在从社会角度估计美国 2019 年非透析依赖性(NDD)CKD 患者因 ESA 注射给药而产生的额外成本。ESA 给药相关的额外成本估计为引入安全性和疗效与 ESA 相当的口服治疗时可避免的年度成本之和。成本构成包括直接医疗保健成本、交通成本以及患者和护理人员(如适用)的工作生产力损失成本。成本是根据科学出版物、政府机构以及最近对美国贫血和 CKD 患者及其护理人员的调查结果估算的。考虑了给药的设定(即在家中还是在诊所)、给药频率以及保险类型。在美国,2019 年因 ESA 给药而产生的年度额外成本估计为 25 亿美元,这是基于接受 ESA 治疗的 462,005 名贫血和 NDD-CKD 患者估算得出的。总体而言,这些成本中有 94.4%(24 亿美元)是因在诊所进行 ESA 给药而产生的。按保险类型对成本进行分层时,医疗保险覆盖的患者占总年度额外成本的 79.4%(20 亿美元)。总年度额外成本的最大贡献者是直接医疗保健成本(14 亿美元,占 54.9%),其次是患者工作生产力损失成本(8.46 亿美元,占 33.9%)、护理人员工作生产力损失成本(1.97 亿美元,占 7.9%)和交通成本(8100 万美元,占 3.3%)。每月接受一次给药的患者每次给药的年度额外成本为 2572 美元,每周接受三次给药的患者每次给药的年度额外成本为 20948 美元,而在家中接受每月给药的患者每次给药的年度额外成本为 1123 美元,每周接受三次给药的患者每次给药的年度额外成本为 2109 美元。在 ESA 给药水平(即每次 ESA 给药),每次诊所 ESA 给药的总额外成本估计为 128 美元,每次家庭 ESA 给药的总额外成本估计为 7 美元,不包括监测成本。NDD-CKD 患者的 ESA 注射给药会带来巨大的经济负担。从社会角度来看,引入口服治疗有潜力带来重要的成本节约。本研究由 Otsuka Pharmaceutical Development & Commercialization, Inc. 和 Akebia Therapeutics, Inc. 资助。研究赞助商参与了研究设计、数据收集、分析、数据解释、报告撰写以及决定提交手稿供发表。Gauthier-Loiselle、Cloutier、Serra、Bungay 和 Guérin 是 Analysis Group, Inc. 的员工,该咨询公司因开展这项研究而从 Otsuka Pharmaceutical Development & Commercialization, Inc. 获得了资金。Michalopoulos 是 Otsuka Pharmaceutical Development & Commercialization, Inc. 在研究期间的员工。Szabo 是 Akebia Therapeutics, Inc. 的员工。