Department of Public Health, School of Public Health, Maragheh University of Medical Sciences, Maragheh, Iran.
Department of Health Management & Economics, School of Public Health, Tehran University of Medical Sciences, Tehran, Iran.
Int J Health Policy Manag. 2022 Jul 1;11(7):1112-1119. doi: 10.34172/ijhpm.2021.04. Epub 2021 Feb 3.
Beta-thalassemia minor and thalassemia major are an autosomal recessive disease with hypochromic, microcytic anemia, and morbidities, Today, therapeutic advances have significantly improved the life expectancy of thalassemia major patients, but at the cost of financial toxicity. The present study aimed to investigate the possibility of increasing the funding for thalassemia screening programs and comparing the cost-effectiveness of screening for thalassemia in the treatment of the patients.
In this study, screening for thalassemia minor was compared with the treatment of thalassemia major patients. A decision tree model was used for analysis. A hospital database, supplemented with a review of published literature, was used to derive input parameters for the model. A lifetime study horizon was used and future costs and consequences were discounted at 3%. The approach of purchases of services was used to evaluate the screening test costs for patients with thalassemia major. Also, a bottom-up method was applied to estimate other screening and treatment costs. All the costs were calculated over one year. The number of gained quality-adjusted life years (QALYs) was calculated using the EQ-5D questionnaire in the evaluated patients.
In this study, 26.97 births of patients with thalassemia major were prevented by screening techniques. On the other hand, total screening costs for patients with thalassemia major were estimated equal to US$ 879879, while the costs of preventing the birth of each thalassaemia major patient was US$ 32 624 by screening techniques. In comparison, the cost of managing a patient with thalassemia major is about US$ 136 532 per year. The life time QALYs for this is 11.8 QALYs. Results are presented using a societal perspective. Incremental cost per QALY gained with screening as compared with managing thalassaemia major was US$ 11 571.
Screening is a long-term value for money intervention that is highly cost effective and its long-term clinical and economic benefits outweigh those of managing thalassaemia major patients.
β-地中海贫血和重型地中海贫血是一种常染色体隐性遗传病，表现为低色素、小细胞性贫血和各种并发症。如今，治疗方法的进步显著提高了重型地中海贫血患者的预期寿命，但这也带来了经济毒性。本研究旨在探讨增加地中海贫血筛查计划资金的可能性，并比较筛查和治疗重型地中海贫血患者的成本效益。
本研究比较了β-地中海贫血筛查与重型地中海贫血患者的治疗。采用决策树模型进行分析。利用医院数据库，并结合文献复习，得出模型的输入参数。采用终生研究时间，并对未来成本和后果贴现 3%。采用购买服务的方法评估重型地中海贫血患者筛查试验的成本。此外，还采用自下而上的方法估算其他筛查和治疗成本。所有成本均按一年计算。采用 EQ-5D 问卷计算评估患者的质量调整生命年（QALY）数。
通过筛查技术，可预防 26.97 例重型地中海贫血患者的出生。另一方面，重型地中海贫血患者的总筛查成本估计为 879879 美元，而通过筛查技术预防每个重型地中海贫血患者出生的成本为 32624 美元。相比之下，管理一名重型地中海贫血患者的成本约为每年 136532 美元。其终生 QALY 为 11.8。结果从社会角度呈现。与管理重型地中海贫血相比，筛查每获得一个 QALY 的增量成本为 11571 美元。
筛查是一种具有长期成本效益的干预措施，非常具有成本效益，其长期的临床和经济效益超过了管理重型地中海贫血患者的效益。
