Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, No.44 Wenhuaxi Road, Jinan, 250012, Shandong, China.
NHC Key Lab of Health Economics and Policy Research, Shandong University), No.44 Wenhuaxi Road, Jinan, 250012, Shandong, China.
Orphanet J Rare Dis. 2023 Aug 29;18(1):252. doi: 10.1186/s13023-023-02858-4.
β-thalassaemia major poses a substantial economic burden, especially in adults. We aimed to estimate the economic burden of adult patients with β-thalassaemia major from a societal perspective using the real-world data. According to the clinical guideline, we also estimated the annual medical costs for patients with the same body weight and calculated the lifetime medical costs over 50 years in mainland China.
This was a retrospective cross-sectional study. An online survey with snowball sampling covering seven provinces was conducted. We extracted patient demographics, caregiver demographics, disease and therapy information, caring burden, and costs for adult patients diagnosed with β-thalassaemia major and their primary caregivers. In the real world, we estimated the annual direct medical cost, direct nonmedical cost, and indirect cost. In addition, we calculated the annual direct medical cost and lifetime direct medical cost by weight with discounted and undiscounted rates according to the clinical guideline.
Direct medical costs was the main driver of total cost, with blood transfusion and iron chelation therapy as the most expensive components of direct medical cost. In addition, adult patients with β-thalassaemia major weighing 56 kg were associated with an increase of $2,764 in the annual direct medical cost using the real-world data. The undiscounted and discounted (5% discount rate) total lifetime treatment costs were $518,871 and $163,441, respectively.
Patients with β-thalassaemia major often encounter a substantial economic burden in mainland China. Efforts must be made to help policymakers develop effective strategies to reduce the burden and pevalence of thalassaemia.
重型β地中海贫血症给患者带来了巨大的经济负担，尤其是成年患者。本研究旨在从社会角度出发，利用真实世界数据来评估成年重型β地中海贫血症患者的经济负担。根据临床指南，我们还估算了相同体重患者的年度医疗费用，并计算了中国大陆患者 50 年内的终身医疗费用。
这是一项回顾性的横断面研究。采用滚雪球抽样方法，对七个省份的成年重型β地中海贫血症患者及其主要照顾者进行了一项在线调查。我们提取了患者和照顾者的人口统计学资料、疾病和治疗信息、照顾负担以及医疗费用。在真实世界中，我们估算了直接医疗成本、直接非医疗成本和间接成本。此外，我们根据临床指南，按体重计算了折现率和不折现率下的年度直接医疗费用和终身直接医疗费用。
直接医疗费用是总费用的主要驱动因素，其中输血和铁螯合治疗是直接医疗费用中最昂贵的部分。此外，使用真实世界数据，体重为 56kg 的成年重型β地中海贫血症患者的年度直接医疗费用增加了 2764 美元。未折现和折现（5%折扣率）的终身总治疗费用分别为 518871 美元和 163441 美元。
中国大陆的重型β地中海贫血症患者往往面临着巨大的经济负担。必须努力帮助政策制定者制定有效的策略来减轻地中海贫血症的负担和发病率。
