title: PAP assays in newborn screening for cystic fibrosis: a population-based cost-effectiveness study.
abstract: To compare the cost effectiveness of adding a pancreatitis-associated protein (PAP) assay to common immunoreactive trypsinogen (IRT) and DNA cystic fibrosis (CF) newborn screening strategies.
  
  
  
  Using data collected on 553,167 newborns, PAP cut-offs were calculated based on non-inferiority of the detection rates of classical forms of CF. Cost effectiveness was considered from the third-party payer's perspective using only direct medical costs, and the unit costs of PAP assays were assessed based on a micro-costing study. Robustness of the cost-effectiveness estimates was assessed, taking the secondary outcomes of screening (ie. detecting mild forms and CF carriers) into account.
  
  
  
  IRT/DNA, IRT/PAP, and IRT/PAP/DNA strategies had similar detection rates for classical forms of CF, but the strategies involving PAP assays detected smaller numbers of mild forms of CF. The IRT/PAP strategy was cost-effective in comparison with either IRT/DNA or IRT/PAP/DNA. IRT/PAP/DNA screening was cost-effective in comparison with IRT/DNA if relatively low value was assumed to be attached to the identification of CF carriers.
  
  
  
  IRT/PAP strategies could be strictly cost-effective, but dropping DNA would mean the test could not detect CF carriers. IRT/PAP/DNA strategies could be a viable option as they are significantly less costly than IRT/DNA, but still allow CF carrier detection.
authors: INSERM, UMR912 "Economics and Social Sciences Applied to Health & Analysis of Medical Information" (SESSTIM), 13006, Marseille, France Aix Marseille University, UMR_S912, IRD, 13006, Marseille, France valerie.seror@inserm.fr.
authors: Valerie
authors: V
authors: Seror
authors: INSERM, UMR912 "Economics and Social Sciences Applied to Health & Analysis of Medical Information" (SESSTIM), 13006, Marseille, France Aix Marseille University, UMR_S912, IRD, 13006, Marseille, France.
authors: Caroline
authors: C
authors: Cao
authors: Hôpital sud CHU Université de Rennes I, 35203 Rennes, France Association Française pour le Dépistage et la Prévention des Handicaps de l'Enfant (AFDPHE), 75015, Paris, France.
authors: Michel
authors: M
authors: Roussey
authors: INSERM, UMR912 "Economics and Social Sciences Applied to Health & Analysis of Medical Information" (SESSTIM), 13006, Marseille, France Aix Marseille University, UMR_S912, IRD, 13006, Marseille, France.
authors: Roch
authors: R
authors: Giorgi
mesh_terms: D000951:Antigens, Neoplasm / Q000097:blood*; D014408:Biomarkers, Tumor / Q000097:blood*; D003362:Cost-Benefit Analysis; D003550:Cystic Fibrosis / Q000097:blood / Q000175:diagnosis* / Q000191:economics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D005260:Female; D005602:France; D006801:Humans; D007231:Infant, Newborn; D037181:Lectins, C-Type / Q000097:blood*; D008297:Male; D015997:Neonatal Screening / Q000191:economics* / Q000379:methods; D000073718:Pancreatitis-Associated Proteins; D011446:Prospective Studies; D012680:Sensitivity and Specificity; D014362:Trypsinogen / Q000097:blood
chemical_list: D000951:Antigens, Neoplasm; D014408:Biomarkers, Tumor; C505032:CFTR protein, human; D037181:Lectins, C-Type; D000073718:Pancreatitis-Associated Proteins; C000612141:REG3A protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D014362:Trypsinogen
keywords: Newborn screening; PAP assay; cost-effectiveness; cystic fibrosis; micro-costing