European countries. Methods: This was a cross-sectional study carried out in the United Kingdom, France and Germany. Data was collected from July 2015 to November 2015. Healthcare costs (hospitalizations, emergencies, medical tests, drugs consumption, visits to GPs and specialists, health material and healthcare transport) and non-healthcare costs (social services and informal care) were identified and valued (reference year: 2014). EQ-5D instrument, Barthel index and Zarit score were also used to reflect the burden and the social impact of the disease beyond the healthcare cost. Results: 86 children with SMA were included, 26.7% of them were Type I and 73.3% were Type II or III. The annual average costs associated with SMA reached 54,295V in UK, 32,042V in France and 52,985V in Germany. Direct non-healthcare costs ranged between 65%-80% of the total cost associated with SMA disease in the three countries analysed. More precisely, the cost associated with informal care was 40,526V (74% of the total cost) for UK, 26,619V (80% of the total cost) for France and 39,926V (77% of the total cost) for Germany. Regarding HRQOL, it was observed that in France and United Kingdom children have a very poor quality of life (0.11 and 0.16, respectively, using time trade-off (TTO) utility score). On contrary, German children had a significantly better quality of life with a TTO of 0.53. Conclusions: SMA represents a considerably high socioeconomic impact both in terms of healthcare and social costs and in reduced HRQOL of children affected. Our analysis may help to design more efficient and equitable policies, with special emphasis on the need to increase the resources and the support provided to the families.
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