International Perspectives on the Cost-Effectiveness of Tandem Mass Spectrometry for Rare Metabolic Conditions

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 forcomparability and transferability of results across settings.Results: The primary outcome measure was usually life years gained (LYG) or quality adjusted lifeyears 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.