Patient characteristics and hospitalisation costs of spinal muscular atrophy in Spain: a retrospective multicentre database analysis

Abstract

ObjectivesTo analyse the characteristics of patients diagnosed with spinal muscular atrophy in Spain, and to revise data on disease management and use of resources in both public and private healthcare centres.DesignA retrospective multicentre database analysis.Setting870 admission records registered between 1997 and 2015 with a diagnosis of spinal muscular atrophy were extracted from a Spanish claims database that includes hospital inpatient and outpatient admissions from 313 public and 192 private hospitals in Spain.ResultsAdmission files corresponded to 705 patients; 61.99% were males and 38.01% females. Average patient age was 37 years. Disease comorbidities registered during the admission consistently included hypertension, scoliosis and respiratory failures, all associated with the standard disease course. Regarding disease management at the hospital level, patients were mostly admitted through scheduled appointments (58.16%), followed by emergency admissions (41.72%), and into neurology services in 17% of the cases. Mean hospitalisation time was 10.45 days and in-hospital mortality reached 5.29%. The overall direct medical costs of spinal muscular atrophy were €291 525, excluding medication. The average annual cost per admission was €6274, with large variations likely to reflect disease complexity and that increases with length of stay.ConclusionsThe rarity of the disease difficulties the study of demographics and management; yet, an analysis of patient characteristics provides necessary information that can be used by governments to establish more efficient healthcare protocols. This study reflects the impact that individual needs and disease severity can have in disease burden calculations. Forthcoming decision-making policies should take into account medical costs and its variability, as well as pharmaceutical expenses and indirect costs. To our knowledge, this is the first study evaluating the use of healthcare resources of patients with spinal muscular atrophy in Spain.

Costs of Illness of Spinal Muscular Atrophy: A Systematic Review

“…Of these, 289 were duplicates, 665 were excluded following title and abstract screening, and 29 were selected for fulltext review. Finally, 14 articles [13][14][15][16][17][18][19][20][21][22][23][24][25][26] were considered for data extraction and synthesis. The primary reason for article exclusion was lack of relevant cost data.…”

“…Nine (64%) studies reported estimates of costs of SMA type I [ 15 – 18 , 20 – 23 , 25 ], four (29%) of costs of SMA type II [ 13 , 20 , 22 , 25 ], four (29%) of costs of SMA type III [ 15 , 20 , 22 , 25 ], one (7%) of costs of SMA type IV [ 21 ], and one (7%) study of costs of progressive SMA, other SMA, and unspecified SMA [ 15 ]. Eleven (79%) studies did not explicitly disclose SMA type, or reported pooled results for more than one type of SMA (in some cases in addition to estimates stratified by SMA type, as reported above) [ 13 – 15 , 18 – 21 , 23 – 26 ]. Two studies (14%) estimated costs of “infantile SMA”, “childhood-onset SMA”, and/or “late-onset SMA” [ 21 , 23 ], for the purpose of this review approximated as SMA type I, SMA type II/III, and SMA type IV, respectively.…”

“…In total, nine (64%) studies estimated the per-patient annual direct medical cost of SMA [13,[18][19][20][21][22][23][24]26], five (36%) the per-patient annual direct non-medical cost [13,20,22,23,26], four (29%) the per-patient annual indirect cost [20,22,23,25], and six (43%) studies the per-patient annual total cost of illness [13,20,22,23,25,26] (Table 1). Additionally, three (21%) studies estimated the per-patient cost per hospitalization of SMA [14][15][16], and one (7%) the perpatient annual hospitalization cost of SMA [17]. Finally, Chambers et al [25] estimated the per-patient annual direct cost of SMA (comprising both medical and non-medical costs).…”

“…When accounting for the cost of nusinersen, the per-patient annual direct medical cost was estimated at $1,000,280 (SMA type I) and $1,109,060 (SMA types II-IV) in the USA [18]. As shown in Table 3 Three studies [14][15][16] examined the per-patient cost per hospitalization of SMA (Table 2). Cardenas et al [16] estimated the mean per-patient cost per hospitalization (derived from total hospital charges) at $54,890 for 237 US patients (43% male, distribution of age not reported) with SMA type I.…”

Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era

“…Beyond core symptoms (e.g. respiratory, motor function), clinical issues that can be more prominent in adults include excessive fatigue [10], kidney stones, osteoporosis, acidosis [11], hypertension [12], weight management, sexual health and hormonal issues. Often, adults also receive inadequate reproductive health advice, for example, being told that having a child is not possible/too dangerous despite many adults having uneventful pregnancies [13].…”