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Logo of bmcpediBioMed Centralsearchsubmit a manuscriptregisterthis articleBMC Pediatrics
 
BMC Pediatr. 2012; 12: 60.
Published online Jun 8, 2012. doi:  10.1186/1471-2431-12-60
PMCID: PMC3464722
Cost-effectiveness analysis of universal newborn screening for medium chain acyl-CoA dehydrogenase deficiency in France
Françoise F Hamerscorresponding author1 and Catherine Rumeau-Pichon1
1Department of Economic and Public Health Evaluation, Haute Autorité de Santé (HAS), 2 avenue du Stade de France, Saint-Denis, France
corresponding authorCorresponding author.
Françoise F Hamers: f.hamers/at/has-sante.fr; Catherine Rumeau-Pichon: c.rumeaupichon/at/has-sante.fr
Received January 10, 2012; Accepted June 8, 2012.
Abstract
Background
Five diseases are currently screened on dried blood spots in France through the national newborn screening programme. Tandem mass spectrometry (MS/MS) is a technology that is increasingly used to screen newborns for an increasing number of hereditary metabolic diseases. Medium chain acyl-CoA dehydrogenase deficiency (MCADD) is among these diseases. We sought to evaluate the cost-effectiveness of introducing MCADD screening in France.
Methods
We developed a decision model to evaluate, from a societal perspective and a lifetime horizon, the cost-effectiveness of expanding the French newborn screening programme to include MCADD. Published and, where available, routine data sources were used. Both costs and health consequences were discounted at an annual rate of 4%. The model was applied to a French birth cohort. One-way sensitivity analyses and worst-case scenario simulation were performed.
Results
We estimate that MCADD newborn screening in France would prevent each year five deaths and the occurrence of neurological sequelae in two children under 5 years, resulting in a gain of 128 life years or 138 quality-adjusted life years (QALY). The incremental cost per year is estimated at €2.5 million, down to €1 million if this expansion is combined with a replacement of the technology currently used for phenylketonuria screening by MS/MS. The resulting incremental cost-effectiveness ratio (ICER) is estimated at €7 580/QALY. Sensitivity analyses indicate that while the results are robust to variations in the parameters, the model is most sensitive to the cost of neurological sequelae, MCADD prevalence, screening effectiveness and screening test cost. The worst-case scenario suggests an ICER of €72 000/QALY gained.
Conclusions
Although France has not defined any threshold for judging whether the implementation of a health intervention is an efficient allocation of public resources, we conclude that the expansion of the French newborn screening programme to MCADD would appear to be cost-effective. The results of this analysis have been used to produce recommendations for the introduction of universal newborn screening for MCADD in France.
Keywords: Medium-chain Acyl-CoA dehydrogenase deficiency, Cost effectiveness, Neonatal screening, Health policy, Tandem mass spectrometry, France
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