Cost-effectiveness of newborn screening for cystic fibrosis determined with real-life data

Background: Previous cost-effectiveness studies using data from the literature showed that newborn screening for cystic fibrosis (NBSCF) is a good economic option with positive health effects and longer survival. Methods: We used primary data to compare cost-effectiveness of four screening strategies for NBSCF, i.e. immunoreactive trypsinogen-testing followed by pancreatitis-associated protein-testing (IRT-PAP), IRT-DNA, IRT-DNA sequencing, and IRT-PAP-DNA sequencing, each compared to no-screening. A previously developed decision analysis model for NBSCF was fed with model parameters mainly based on a study evaluating two novel screening strategies among 145,499 newborns in The Netherlands. Results: The four screening strategies had cost-effectiveness ratios varying from sic23,600 to sic29,200 per life-year gained. IRT PAP had the most favourable cost-effectiveness ratio. Additional life-years can be gained by IRT DNA but against higher costs. When treatment costs reduce with 5% due to early diagnosis, screening will lead to financial savings. Conclusion: NBSCF is as an economically justifiable public health initiative. Of the four strategies tested IRT PAP is the most economic and this finding should be included in any decision making model, when considering implementation of newborn screening for CF. (C) 2014 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.