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Cost-effectiveness of 4 neonatal screening strategies for cystic fibrosis

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Author: Akker-van Marle, M.E. van den · Dankert, H.M. · Verkerk, P.H. · Dankert-Roelse, J.E.
Type:article
Date:2006
Institution: TNO Kwaliteit van Leven
Source:Pediatrics, 3, 118, 896-905
Identifier: 239465
doi: doi:10.1542/peds.2005-2782
Keywords: Health · Jeugd en Gezondheid · Cost-effectiveness · Economic evaluation · Neonatal · Screening-newborn · DNA · Cost effectiveness analysis · Economic aspect · Gel electrophoresis · Gene mutation · Netherlands · Blood · Economics · Evaluation · Genetics · Immunoassay · Methodology · Newborn · Nucleotide sequence · Two dimensional gel electrophoresis · Cost-Benefit Analysis · Cystic Fibrosis · DNA Mutational Analysis · Electrophoresis, Gel, Two-Dimensional · Humans · Immunoassay · Infant, Newborn · Neonatal Screening · Trypsin

Abstract

OBJECTIVES. The purpose of this work was to assess the costs of 4 neonatal screening strategies for cystic fibrosis in relation to health effects. In each strategy, the first test was the measurement of serum concentration of immunoreactive trypsin. The second step consisted of either a second immunoreactive trypsin test (strategy 1) or a multiple mutation analysis (strategy 2). In strategies 3 and 4, a third step was added to strategy 2: a second immunoreactive trypsin test (strategy 3) or an extended mutation analysis of the cystic fibrosis gene, that is, a denaturing gradient gel electrophoresis analysis (strategy 4). METHODS. We conducted an economic-modeling exercise in the Netherlands based on published data and expert opinions. Subjects were a hypothetical cohort of 200 000 neonates, the approximate number of children born annually in the Netherlands, and we assessed the costs and number of life-years gained as a result of neonatal screening for cystic fibrosis. The costs and effects of changes in reproductive decisions because of neonatal screening were also assessed. RESULTS. Immunoreactive trypsin + immunoreactive trypsin had the most favorable cost-effectiveness ratio of €24 800 per life-year gained. Immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis achieved more health effects than immunoreactive trypsin + DNA + immunoreactive trypsin at lower cost. The incremental costs per life-year gained of the immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis strategy compared with the immunoreactive trypsin + immunoreactive trypsin strategy were €130 700, whereas the incremental costs of the immunoreactive trypsin + DNA strategy compared with the immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis strategy were €2 154 300. When changes in reproductive decisions as a result of neonatal screening are also taken into account, neonatal screening for cystic fibrosis may lead to financial savings of approximately €1.8 million annually, depending on the screening strategy used. CONCLUSIONS. Cystic fibrosis screening for neonates is a good economic option, and positive health effects can also be expected. Immunoreactive trypsin + immunoreactive trypsin and immunoreactive trypsin + DNA + denaturing gradient gel electrophoresis are the most cost-effective strategies. Copyright © 2006 by the American Academy of Pediatrics. Chemicals / CAS: DNA, 9007-49-2; trypsin, 9002-07-7; Trypsin, EC 3.4.21.4