Cost-effectiveness analysis of universal newborn screening for medium chain acyl-CoA dehydrogenase deficiency in France Full Text
BMC Pediatrics, 06/21/2012
Hamers FF et al. – Although France has not defined any threshold for judging whether the implementation of a health intervention is an efficient allocation of public resources, they conclude that the expansion of the French newborn screening programme to Medium chain acyl–CoA deficiency (MCADD) would appear to be cost–effective.Methods
- The authors 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.
- The authors 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 euros, down to 1 million euros 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 7580 euros/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.