• Josep Darbà.
• Department of Economics, Universitat de Barcelona, Diagonal 696, 08034, Barcelona, Spain. darba@ub.edu.
• Clin Drug Investig. 2020 Apr 1; 40 (4): 335-341.

BackgroundSpinal muscular atrophy (SMA) is a rare disorder, estimated to affect 1 per 10,000 live births. Patients affected with SMA often require intensive, chronic healthcare, which represents great social and economic costs.ObjectiveThis study aimed to evaluate the direct medical costs of SMA, from the National Health System perspective in Catalonia, and provide regional data for the development of optimal disease management protocols and resource allocation decisions at the regional level.MethodsA retrospective, population-based study was designed based on admission records from primary care centres, hospitals and specialised care settings (inpatient and outpatient care), emergency services and extended care facilities obtained from a regional governmental claims database.ResultsA total of 396 patients met the inclusion criteria. Annual direct medical costs summed €58,606 per patient, taking into account the use of healthcare resources at all levels of care and excluding the cost of prescription medication. Specialised care represented 81% of the expenses that were mostly associated with respiratory manifestations of SMA. In the year 2016, 71.26% of patients with SMA had four or more systems affected by a chronic condition, versus 23.50% in the general population, which had an impact on healthcare use.ConclusionsInpatient extended care and the increased presence of multimorbid chronic conditions in patients with SMA must be taken into account in order to develop multidisciplinary treatment protocols that reflect the complexity of SMA. Forthcoming resource allocation decisions should reflect the intensive use of specialised care registered in patients with SMA.

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