title: Cost-effectiveness of newborn screening for cystic fibrosis determined with real-life data.
abstract: 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 €23,600 to €29,200 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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Child Health, TNO, Leiden, The Netherlands. Electronic address: Kitty.vanderPloeg@tno.nl.
authors.forename: C P B
authors.initials: CP
authors.lastname: van der Ploeg
authors.affiliation: Department of Medical Decision Making, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: M E
authors.initials: ME
authors.lastname: van den Akker-van Marle
authors.affiliation: Department of Research and Innovation, Atrium Medical Center, Heerlen, The Netherlands; Laboratory for Infectious Diseases and Perinatal Screening, RIVM, Bilthoven, The Netherlands.
authors.forename: A M M
authors.initials: AM
authors.lastname: Vernooij-van Langen
authors.affiliation: Laboratory for Infectious Diseases and Perinatal Screening, RIVM, Bilthoven, The Netherlands.
authors.forename: L H
authors.initials: LH
authors.lastname: Elvers
authors.affiliation: Department of Clinical Genetics, VU University Medical Center, Amsterdam, The Netherlands.
authors.forename: J J P
authors.initials: JJ
authors.lastname: Gille
authors.affiliation: Department of Child Health, TNO, Leiden, The Netherlands.
authors.forename: P H
authors.initials: PH
authors.lastname: Verkerk
authors.affiliation: Department of Pediatrics, Atrium Medical Center, Heerlen, The Netherlands.
authors.forename: J E
authors.initials: JE
authors.lastname: Dankert-Roelse
mesh_terms: D000951:Antigens, Neoplasm / Q000032:analysis / Q000235:genetics; D014408:Biomarkers, Tumor / Q000032:analysis / Q000235:genetics; D003362:Cost-Benefit Analysis; D003550:Cystic Fibrosis / Q000175:diagnosis / Q000191:economics / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000032:analysis / Q000235:genetics; D003661:Decision Support Techniques; D005820:Genetic Testing / Q000191:economics / Q000379:methods; D006801:Humans; D007231:Infant, Newborn; D037181:Lectins, C-Type / Q000032:analysis / Q000235:genetics; D009154:Mutation; D015997:Neonatal Screening / Q000191:economics / Q000458:organization & administration; D009426:Netherlands; D000073718:Pancreatitis-Associated Proteins; D012680:Sensitivity and Specificity; D014362:Trypsinogen / Q000032:analysis / Q000235:genetics
chemical_list: D000951:Antigens, Neoplasm; D014408:Biomarkers, Tumor; D037181:Lectins, C-Type; D000073718:Pancreatitis-Associated Proteins; C000612141:REG3A protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D014362:Trypsinogen
keywords: Cost-effectiveness; Cystic fibrosis; Newborn screening