Author:

Keywords:

Science & Technology, Life Sciences & Biomedicine, Respiratory System, Cystic fibrosis, Newborn screening, Health economic evaluation, Systematic review, COST-EFFECTIVENESS, SURVIVAL, IMPACT, Cost-Benefit Analysis, Cystic Fibrosis, Humans, Infant, Newborn, Neonatal Screening, 1103 Clinical Sciences, 3202 Clinical sciences

Abstract:

BACKGROUND: Early detection of cystic fibrosis through newborn screening has significant clinical benefits. Cost-effectiveness plays an important role in selecting the optimal screening strategy from the many available options. OBJECTIVES: The objectives of this study are (1) to summarize study estimates of cost-effectiveness of cystic fibrosis newborn screening (CFNBS) strategies as compared to other strategies, (2) to assess the quality of the studies identified, and (3) to identify determinants of cost-effectiveness. METHODS: Electronic databases were searched from 2007 to June 2017. Health economic evaluations describing the cost-effectiveness of two or more CFNBS strategies were included. RESULTS: Six health economic evaluations were found. Where included in the comparison, IRT/PAP consistently was the most cost-effective strategy in terms of cost per case detected or life years gained. However, some heterogeneity with respect to cut-off values used and the number of DNA mutations included in the screening strategies was observed, and the methodological quality differed considerably between studies. CONCLUSIONS: The evidence suggested that (i) all screening strategies are cost-effective as compared to the no-screening option and (ii) IRT-PAP seems to be the most cost-effective screening strategy towards CFNBS. Methodological and contextual differences of the individual studies make it difficult to derive strong conclusions from this evidence. Nevertheless, from a health-economic perspective, IRT-PAP should be included as an alternative when deciding on the screening strategy in the implementation of CFNBS.