Sims Erika J, Mugford Miranda, Clark Allan, Aitken David, McCormick Jonathan, Mehta Gita, Mehta Anil
UK Cystic Fibrosis Database, Division of Maternal and Child Health Sciences, Ninewells Hospital and Medical School, University of Dundee, Dundee, UK.
Lancet. 2007 Apr 7;369(9568):1187-95. doi: 10.1016/S0140-6736(07)60565-0.
Newborn screening for cystic fibrosis might not be introduced if implementation and running costs are perceived as prohibitive. Compared with clinical diagnosis, newborn screening is associated with clinical benefit and reduced treatment needs. We estimate the potential savings in treatment costs attributable to newborn screening.
Using the UK Cystic Fibrosis Database, we used a prevalence strategy to undertake a cost of illness retrospective snapshot cohort study. We estimated yearly costs of long-term therapies and intravenous antibiotics for 184 patients who were diagnosed as a result of screening as newborn babies, and 950 patients who were clinically diagnosed aged 1-9 years in 2002. Costs of adding cystic fibrosis screening to an established newborn screening service in Scotland were adjusted to 2002 prices and applied to the UK as a whole. Costs were recalculated in US$.
Cost of therapy for patients diagnosed by newborn screening was significantly lower than equivalent therapies for clinically diagnosed patients: mean ($7228 vs $12 008, 95% CI of difference -6736 to -2028, p<0.0001) and median ($352 vs $2442, -1916 to -180, p<0.0001). When we limited the clinically diagnosed group to only those diagnosable with a 31 cystic fibrosis transmembrane regulator mutation assay and assumed similar disease progression in the clinically diagnosed group as in the newborn screening group, we showed that mean ($3,397,344) or median ($947,032) drug cost savings could have offset the estimated cost of adding cystic fibrosis to a UK national newborn screening service ($2,971,551).
Including indirect costs savings, newborn screening for cystic fibrosis might have even greater financial benefits to society than our estimate shows. Clinical, social, and now economic evidence suggests that universal newborn screening programmes for cystic fibrosis should be adopted internationally.
如果实施和运营成本过高，可能不会开展新生儿囊性纤维化筛查。与临床诊断相比，新生儿筛查具有临床益处且可减少治疗需求。我们估算了新生儿筛查在治疗成本方面可能节省的费用。
利用英国囊性纤维化数据库，我们采用患病率策略进行了一项疾病成本回顾性快照队列研究。我们估算了184例新生儿期筛查确诊患者以及950例2002年临床诊断为1 - 9岁患者的长期治疗及静脉用抗生素的年度费用。将在苏格兰现有新生儿筛查服务中增加囊性纤维化筛查的成本调整为2002年价格，并应用于整个英国。成本重新换算为美元。
新生儿筛查确诊患者的治疗成本显著低于临床诊断患者的同等治疗成本：均值（7228美元对12008美元，差值的95%置信区间为 - 6736至 - 2028，p<0.0001）和中位数（352美元对2442美元， - 1916至 - 180，p<0.0001）。当我们将临床诊断组仅限定为那些可通过囊性纤维化跨膜调节因子突变检测确诊的患者，并假设临床诊断组与新生儿筛查组疾病进展相似时，我们发现均值（3397344美元）或中位数（947032美元）的药物成本节省可抵消在英国全国新生儿筛查服务中增加囊性纤维化筛查的估计成本（2971551美元）。
包括间接成本节省在内，新生儿囊性纤维化筛查对社会的经济效益可能比我们的估计更大。临床、社会以及现在的经济证据表明，国际上应采用针对囊性纤维化的普遍新生儿筛查项目。
