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

ObjectivesTo 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.MethodsA 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.ResultsThe 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.ConclusionsDifferences 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.