罕见病的社会经济成本与经济困难风险：一项横断面研究。
Socio-economic costs of rare diseases and the risk of financial hardship: a cross-sectional study.
作者信息
Chung Claudia C Y, Ng Nicole Y T, Ng Yvette N C, Lui Adrian C Y, Fung Jasmine L F, Chan Marcus C Y, Wong Wilfred H S, Lee So Lun, Knapp Martin, Chung Brian H Y
机构信息
Department of Paediatrics and Adolescent Medicine, School of Clinical Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
出版信息
Lancet Reg Health West Pac. 2023 Feb 23;34:100711. doi: 10.1016/j.lanwpc.2023.100711. eCollection 2023 May.
BACKGROUND
To achieve universal healthcare coverage (UHC), the rare disease (RD) population must also receive quality healthcare without financial hardship. This study evaluates the impact of RDs in Hong Kong (HK) by estimating cost from a societal perspective and investigating related risk of financial hardship.
METHODS
A total of 284 RD patients and caregivers covering 106 RDs were recruited through HK's largest RD patient group, Rare Disease Hong Kong, in 2020. Resource use data were collected using the Client Service Receipt Inventory for Rare disease population (CSRI-Ra). Costs were estimated using a prevalence-based, bottom-up approach. Risk of financial hardship was estimated using catastrophic health expenditure (CHE) and impoverishing health expenditure (IHE) indicators. Multivariate regression was performed to identify potential determinants.
FINDINGS
Annual total RD costs in HK were estimated at HK$484,256/patient (United States (US) $62,084). Direct non-healthcare cost (HK$193,555/US$24,814) was the highest cost type, followed by direct healthcare (HK$187,166/US$23,995), and indirect (HK$103,535/US$13,273) costs. CHE at the 10% threshold was estimated at 36.3% and IHE at the $3.1 poverty line was 8.8%, both significantly higher than global estimates. Pediatric patients reported higher costs than adult patients (p < 0.001). Longer years since genetic diagnosis was the only factor significantly associated with both total costs (p = 0.026) and CHE (p = 0.003).
INTERPRETATION
This study serves as the first in the Asia Pacific region to simultaneously assess the societal costs and financial hardship related to RDs and highlights the importance of an early genetic diagnosis. These results contribute to existing evidence on the globally ubiquitous high costs of RDs, warranting collaboration between different stakeholders to include RD population in UHC planning.
FUNDING
Health and Medical Research Fund, and the Society for the Relief of Disabled Children.
背景
为实现全民健康覆盖（UHC），罕见病（RD）患者群体也必须在不承受经济困难的情况下获得优质医疗保健。本研究从社会角度估算成本并调查相关经济困难风险，以评估香港（HK）罕见病的影响。
方法
2020年，通过香港最大的罕见病患者群体“香港罕见病联盟”招募了284名涵盖106种罕见病的患者及其照料者。使用罕见病患者群体客户服务收据清单（CSRI-Ra）收集资源使用数据。采用基于患病率的自下而上方法估算成本。使用灾难性卫生支出（CHE）和致贫性卫生支出（IHE）指标估算经济困难风险。进行多变量回归以确定潜在决定因素。
结果
香港罕见病患者的年度总成本估计为每名患者484,256港元（62,084美元）。直接非医疗成本（193,555港元/24,814美元）是最高的成本类型，其次是直接医疗成本（187,166港元/23,995美元）和间接成本（103,535港元/13,273美元）。10%阈值下的灾难性卫生支出估计为36.3%，3.1美元贫困线的致贫性卫生支出为8.8%，两者均显著高于全球估计值。儿科患者的成本高于成人患者（p < 0.001）。自基因诊断以来的时间越长，是唯一与总成本（p = 0.026）和灾难性卫生支出（p = 0.003）均显著相关的因素。
解读
本研究是亚太地区首个同时评估与罕见病相关的社会成本和经济困难的研究，并强调了早期基因诊断的重要性。这些结果为全球普遍存在的罕见病高成本的现有证据做出了贡献，需要不同利益相关者之间开展合作，将罕见病患者群体纳入全民健康覆盖规划。
资助
健康与医学研究基金以及残疾儿童救济协会。
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