Richard Norman1, Marion Haas, Bridget Wilcken. 1. Centre for Health Economics Research and Evaluation, University of Technology, Sydney, Australia. Richard.norman@chere.uts.edu.au
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.
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.
Authors: Yao Ding; John D Thompson; Lisa Kobrynski; Jelili Ojodu; Guisou Zarbalian; Scott D Grosse Journal: J Pediatr Date: 2016-02-11 Impact factor: 4.406
Authors: Maria D Karaceper; Sara D Khangura; Kumanan Wilson; Doug Coyle; Marni Brownell; Christine Davies; Linda Dodds; Annette Feigenbaum; Deshayne B Fell; Scott D Grosse; Astrid Guttmann; Steven Hawken; Robin Z Hayeems; Jonathan B Kronick; Anne-Marie Laberge; Julian Little; Aizeddin Mhanni; John J Mitchell; Meranda Nakhla; Murray Potter; Chitra Prasad; Cheryl Rockman-Greenberg; Rebecca Sparkes; Sylvia Stockler; Keiko Ueda; Hilary Vallance; Brenda J Wilson; Pranesh Chakraborty; Beth K Potter Journal: Orphanet J Rare Dis Date: 2019-03-22 Impact factor: 4.123