Heidari Alireza, Arab Mohammad, Damari Behzad
Health Management and Social Development Research Center, Golestan University of Medical Sciences, Gorgan, Iran.
Department of Management and Health Economic, School of Public Health, Tehran University of Medical Sciences, Tehran, Iran.
Iran J Public Health. 2021 Oct;50(10):2105-2110. doi: 10.18502/ijph.v50i10.7512.
Phenylketonuria (PKU) is an autosomal recessive disorder that screening and timely control of this disorder can prevent the adverse effects. Regarding the high prevalence of PKU in Iran, the PKU screening program was started in Iran in 2006. This study was conducted to determine the cost-effectiveness of PKU screening in Iran.
An economic evaluation was performed among screened and treated individuals compared to no screening in 2013. The study population included 1356132 newborns that screened for PKU diagnosis. Lifetime costs, quality-adjusted life year (QALY) gains and incremental cost-effectiveness ratio (ICER) were calculated from the perspective of government. A discount rate of 3% was considered for both QALYs and costs. A one-way sensitivity analysis was used for assessing the robustness of the results.
The discounted lifetime cost of intervention and non-intervention were $59528953.8 and $85295501.6 respectively. Therefore, the total estimated cost saving was $25766547.84. PKU screening produces an ICER of $1844420 per QALY gained.
Screening and early treatment for PKU is highly cost-effective. Therefore, the screening can improve quality of life of the patients and increase financial saving in health system.
苯丙酮尿症（PKU）是一种常染色体隐性疾病，对该疾病进行筛查并及时控制可预防不良影响。鉴于PKU在伊朗的高发病率，伊朗于2006年启动了PKU筛查项目。本研究旨在确定伊朗PKU筛查的成本效益。
2013年，对已接受筛查和治疗的个体与未进行筛查的个体进行了一项经济评估。研究人群包括1356132名接受PKU诊断筛查的新生儿。从政府角度计算了终身成本、质量调整生命年（QALY）收益和增量成本效益比（ICER）。QALY和成本均采用3%的贴现率。采用单向敏感性分析评估结果的稳健性。
干预组和非干预组的贴现终身成本分别为59528953.8美元和85295501.6美元。因此，估计总成本节约为25766547.84美元。PKU筛查每获得一个QALY的ICER为1844420美元。
PKU筛查和早期治疗具有很高的成本效益。因此，筛查可改善患者的生活质量，并增加卫生系统的财政节约。
