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International perspectives on the cost-effectiveness of tandem mass spectrometry for rare metabolic conditions

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  • Norman, Richard
  • Haas, Marion
  • Wilcken, Bridget

Abstract

Objectives To examine and evaluate the economic evidence regarding the use of tandem mass spectrometry (MS/MS) for the detection of rare metabolic conditions in neonates, and then to consider the transferability of these national-level results to other decision-making contexts.Methods A systematic literature review was undertaken, identifying papers published between January 1997 and March 2008. Thirteen unique cost-effectiveness evaluations were identified and appraised for comparability and transferability of results across settings.Results The primary outcome measure was usually life years gained (LYG) or quality-adjusted life years gained (QALY). The incremental cost-effectiveness ratios (ICER) presented were generally supportive of MS/MS, but showed considerable variation. Differences in assumptions made regarding prevalence and prognosis played a significant role in this variation.Conclusions Differences in study structure, the approach to costing, the choice of intervention, control and outcome measure, and the limit of studies to developed countries makes international generalisation of the cost-effectiveness evidence difficult. The importance of assumptions regarding disease progression and subsequent health care utilisation suggests that further work needs to consider the importance of longer-term follow-up.

Suggested Citation

  • Norman, Richard & Haas, Marion & Wilcken, Bridget, 2009. "International perspectives on the cost-effectiveness of tandem mass spectrometry for rare metabolic conditions," Health Policy, Elsevier, vol. 89(3), pages 252-260, March.
    • Handle: RePEc:eee:hepoli:v:89:y:2009:i:3:p:252-260
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    References listed on IDEAS

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    1. Koopmanschap, Marc A. & Rutten, Frans F. H. & van Ineveld, B. Martin & van Roijen, Leona, 1995. "The friction cost method for measuring indirect costs of disease," Journal of Health Economics, Elsevier, vol. 14(2), pages 171-189, June.
    2. Radhakrishnan, Muralikrishnan & van Gool, Kees & Hall, Jane & Delatycki, Martin & Massie, John, 2008. "Economic evaluation of cystic fibrosis screening: A review of the literature," Health Policy, Elsevier, vol. 85(2), pages 133-147, February.
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    1. Katharina Elisabeth Fischer & Wolf Henning Rogowski, 2014. "Funding Decisions for Newborn Screening: A Comparative Review of 22 Decision Processes in Europe," IJERPH, MDPI, vol. 11(5), pages 1-28, May.
    2. Gillner, Sandra & Gumus, Gulcin & Gross, Edith & Iskrov, Georgi & Raycheva, Ralitsa & Stefanov, Georgi & Stefanov, Rumen & Chalandon, Anne-Sophie & Granados, Alicia & Nam, Julian & Clemens, Andreas & , 2024. "The modernisation of newborn screening as a pan-European challenge – An international delphi study," Health Policy, Elsevier, vol. 149(C).
    3. Kittiphong Thiboonboon & Pattara Leelahavarong & Duangrurdee Wattanasirichaigoon & Nithiwat Vatanavicharn & Pornswan Wasant & Vorasuk Shotelersuk & Suthipong Pangkanon & Chulaluck Kuptanon & Sumonta C, 2015. "An Economic Evaluation of Neonatal Screening for Inborn Errors of Metabolism Using Tandem Mass Spectrometry in Thailand," PLOS ONE, Public Library of Science, vol. 10(8), pages 1-25, August.

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