Shih Sophy T F, Keller Elena, Wiley Veronica, Wong Melanie, Farrar Michelle A, Chambers Georgina M
Surveillance, Evaluation and Research Program, Kirby Institute, University of New South Wales, Sydney, NSW 2052, Australia.
National Perinatal Epidemiology and Statistics Unit, Centre for Big Data Research in Health and School of Clinical Medicine, University of New South Wales, Sydney, NSW 2052, Australia.
Int J Neonatal Screen. 2022 Jul 20;8(3):44. doi: 10.3390/ijns8030044.
Evidence on the cost-effectiveness of newborn screening (NBS) for severe combined immunodeficiency (SCID) in the Australian policy context is lacking. In this study, a pilot population-based screening program in Australia was used to model the cost-effectiveness of NBS for SCID from the government perspective. Markov cohort simulations were nested within a decision analytic model to compare the costs and quality-adjusted life-years (QALYs) over a time horizon of 5 and 60 years for two strategies: (1) NBS for SCID and treat with early hematopoietic stem cell transplantation (HSCT); (2) no NBS for SCID and treat with late HSCT. Incremental costs were compared to incremental QALYs to calculate the incremental cost-effectiveness ratios (ICER). Sensitivity analyses were performed to assess the model uncertainty and identify key parameters impacting on the ICER. In the long-term over 60 years, universal NBS for SCID would gain 10 QALYs at a cost of US $0.3 million, resulting in an ICER of US$33,600/QALY. Probabilistic sensitivity analysis showed that more than half of the simulated ICERs were considered cost-effective against the common willingness-to-pay threshold of A$50,000/QALY (US$35,000/QALY). In the Australian context, screening for SCID should be introduced into the current NBS program from both clinical and economic perspectives.
在澳大利亚的政策背景下，缺乏关于新生儿筛查（NBS）用于重症联合免疫缺陷（SCID）的成本效益的证据。在本研究中，澳大利亚一项基于人群的试点筛查项目被用于从政府角度模拟NBS用于SCID的成本效益。马尔可夫队列模拟嵌套在一个决策分析模型中，以比较两种策略在5年和60年时间范围内的成本和质量调整生命年（QALY）：（1）对SCID进行NBS并采用早期造血干细胞移植（HSCT）治疗；（2）不对SCID进行NBS并采用晚期HSCT治疗。将增量成本与增量QALY进行比较，以计算增量成本效益比（ICER）。进行敏感性分析以评估模型的不确定性，并确定影响ICER的关键参数。在60年以上的长期内，对SCID进行普遍NBS将以30万美元的成本获得10个QALY，导致ICER为33,600美元/QALY。概率敏感性分析表明，超过一半的模拟ICER相对于50,000澳元/QALY（35,000美元/QALY）的常见支付意愿阈值被认为具有成本效益。在澳大利亚的背景下，从临床和经济角度来看，应将SCID筛查纳入当前的NBS项目。
