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美国常染色体显性多囊肾病的社会经济负担。

The societal economic burden of autosomal dominant polycystic kidney disease in the United States.

机构信息

Analysis Group, Inc., Montréal, QC, Canada.

Otsuka Pharmaceutical Development & Commercialization, Inc., Princeton, NJ, USA.

出版信息

BMC Health Serv Res. 2020 Feb 18;20(1):126. doi: 10.1186/s12913-020-4974-4.

Abstract

BACKGROUND

Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common inherited kidney diseases characterized by progressive development of renal cysts and numerous extra-renal manifestations, eventually leading to kidney failure. Given its chronic and progressive nature, ADPKD is expected to carry a substantial economic burden over the course of the disease. However, there is a paucity of evidence on the impact of ADPKD from a societal perspective. This study aimed to estimate the direct and indirect costs associated with ADPKD in the United States (US).

METHODS

A prevalence-based approach using data from scientific literature, and governmental and non-governmental organizations was employed to estimate direct healthcare costs (i.e., medical services, prescription drugs), direct non-healthcare costs (i.e., research and advocacy, donors/recipients matching for kidney transplants, transportation to/from dialysis centers), and indirect costs (i.e., patient productivity loss from unemployment, reduced work productivity, and premature mortality, caregivers' productivity loss and healthcare costs). The incremental costs associated with ADPKD were calculated as the difference between costs incurred over a one-year period by individuals with ADPKD and the US population. Sensitivity analyses using different sources and assumptions were performed to assess robustness of estimates and account for variability in published estimates.

RESULTS

The estimated total annual costs attributed to ADPKD in 2018 ranged from $7.3 to $9.6 billion in sensitivity analyses, equivalent to $51,970 to $68,091 per individual with ADPKD. In the base scenario, direct healthcare costs accounted for $5.7 billion (78.6%) of the total $7.3 billion costs, mostly driven by patients requiring renal replacement therapy ($3.2 billion; 43.3%). Indirect costs accounted for $1.4 billion (19.7%), mostly driven by productivity loss due to unemployment ($784 million; 10.7%) and reduced productivity at work ($390 million; 5.3%). Total excess direct non-healthcare costs were estimated at $125 million (1.7%).

CONCLUSIONS

ADPKD carries a considerable economic burden, predominantly attributed to direct healthcare costs, the majority of which are incurred by public and private healthcare payers. Effective and timely interventions to slow down the progression of ADPKD could substantially reduce the economic burden of ADPKD.

摘要

背景

常染色体显性多囊肾病(ADPKD)是最常见的遗传性肾脏疾病之一,其特征为肾囊肿进行性发展和众多肾脏外表现,最终导致肾衰竭。鉴于其慢性和进行性的性质,ADPKD 在疾病过程中预计会带来巨大的经济负担。然而,从社会角度来看,ADPKD 的影响证据很少。本研究旨在估计美国(US)ADPKD 的直接和间接成本。

方法

采用基于患病率的方法,利用科学文献、政府和非政府组织的数据,估计直接医疗保健成本(即医疗服务、处方药物)、直接非医疗保健成本(即研究和宣传、肾脏移植供体/受体匹配、往返透析中心的交通费用)和间接成本(即失业导致的患者生产力损失、工作生产力降低和过早死亡、照顾者的生产力损失和医疗保健成本)。ADPKD 相关的增量成本是通过计算患有 ADPKD 的个体在一年内的成本与美国人口的成本之间的差异来计算的。使用不同的来源和假设进行敏感性分析,以评估估计的稳健性并考虑已发表估计值的可变性。

结果

2018 年,ADPKD 归因于估计的年度总成本在敏感性分析中从 7.3 亿美元到 9.6 亿美元不等,相当于每个 ADPKD 患者 51970 美元到 68091 美元。在基础方案中,直接医疗保健成本占 7.3 亿美元总成本的 5.7 亿美元(78.6%),主要由需要肾脏替代治疗的患者驱动(3.2 亿美元;43.3%)。间接成本占 1.4 亿美元(19.7%),主要由失业导致的生产力损失驱动(7840 万美元;10.7%)和工作生产力降低驱动(3900 万美元;5.3%)。总超额直接非医疗保健成本估计为 1.25 亿美元(1.7%)。

结论

ADPKD 带来了相当大的经济负担,主要归因于直接医疗保健成本,其中大部分由公共和私人医疗保健支付者承担。有效和及时的干预措施以减缓 ADPKD 的进展可能会大大减轻 ADPKD 的经济负担。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a997/7029467/6e62ffbdc8af/12913_2020_4974_Fig1_HTML.jpg

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