Development of a decision-analytic model for the economic evaluation of newborn screening for spinal muscular atrophy

Economic considerations are increasingly important to help decision makers to efficiently allocate health care resources. To date, very little information is available on the cost-effectiveness of spinal muscular atrophy (SMA) treatment and newborn screening; yet more and more screening programs are being implemented in the USA and in Europe. The aim of this study is therefore to develop a decision-analytic model to assess the cost-effectiveness of newborn screening and pre-symptomatic treatment of SMA as compared to post-symptomatic treatment. Newborn screening for SMA has started since Marsh 05th in Southern Belgium. We are developing a decision-analytic model in Excel with a lifetime horizon and a societal perspective. This model includes events (such as death, permanent ventilation, etc), transition, utilities, and costs. Data was retrieved from literature reviews, available non-published data from Nurture, and data collected from the European Natural History Study in SMA. We also collected data from the large post symptomatic treated cohort of Liege, Belgium. All such data was acquired in centers applying updated standards of care. The quality of life of patients and parents was included in the analysis, as well as the productivity losses of parents. The model thus estimates the costs and effects (expressed in quality-adjusted life years) of newborn screening and to compare it with a situation where all patients are diagnosed post-symptomatically. Initial data suggests promising results and reveal key factors on the cost-effectiveness of NBS. The model requires adjustment to be fully validated.