Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain

Abstract

BackgroundThe aim of this study was to determine the economic burden and health-related quality of life (HRQOL) of patients with Spinal Muscular Atrophy (SMA) and their caregivers in Spain.MethodsThis was a cross-sectional and retrospective study of patients diagnosed with SMA in Spain. We adopted a bottom up, prevalence approach design to study patients with SMA. The patient’s caregivers completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and non-healthcare services. Costs were estimated from a societal perspective (including healthcare costs and non-healthcare costs), and health-related quality of life (HRQOL) was assessed using the EQ-5D questionnaire. The main caregivers also answered a questionnaire on their characteristics and on their HRQOL.ResultsA total of 81 caregivers of patients with different subtypes of SMA completed the questionnaire. Based on the reference unitary prices for 2014, the average annual costs per patient were € 33,721. Direct healthcare costs were € 10,882 (representing around 32.3% of the total cost) and the direct non-healthcare costs were € 22,839 (67.7% of the total cost). The mean EQ-5D social tariff score for patients was 0.16, and the mean score of the EQ-5D visual analogue scale was 54. The mean EQ-5D social tariff score for caregivers was 0.49 and their mean score on the EQ-5D visual analogue scale was 69.ConclusionThe results highlight the burden that SMA has in terms of costs and decreased HRQOL, not only for patients but also for their caregivers. In particular, the substantial social/economic burden is mostly attributable to the high direct non-healthcare costs.

Cost Effectiveness of Nusinersen in the Treatment of Patients with Infantile-Onset and Later-Onset Spinal Muscular Atrophy in Sweden

“…Resource use data for the models were collected from interviews with two Swedish clinical experts, as the economic literature review (Table S1 and Fig. S1 of the ESM) presented resource use and costs from only a German study [57] and a Spanish study [58]. In accordance with Swedish guidelines for health economic evaluations, the societal perspective was applied, including caregiver production loss for one caregiver in accordance with clinical expert input.…”

Economic burden of spinal muscular atrophy in the United States: a contemporary assessment

“…Assessments of the real-world economic burden of SMA, including the healthcare resource utilization (HRU) and healthcare costs associated with the disease, remain limited in the US [11][12][13][14][15][16][17][18][19] . In a study conducted by Dabbous et al 13 , which evaluated the economic burden of patients with SMA1 in the US, healthcare costs in the first month following SMA diagnosis averaged $27,063 per patient compared to $274 among matched patients without SMA.…”

Preference-based measures of health-related quality of life in congenital mobility impairment: a systematic review of validity and responsiveness

“…Fourteen studies included data relating to CP [22, 33, 35, 36, 39, 46, 48-51, 53, 55, 57, 58], six included data relating to SB [21,38,51,52,54,56] and five included data relating to childhood hydrocephalus [40][41][42][43]45]. Data for a range of other relevant conditions were also found in either single studies or from extractable subgroups, these included muscular dystrophy [34,44], spinal muscular atrophy [47], Morquio A syndrome [37], congenital clubfoot [59] and microcephaly [51]. Three studies focused on multiple conditions where sub-group data could not be examined separately [11,15,51].…”

“…In terms of use of PBMs, 22 of the studies used a version of the HUI [15, 21, 22, 33, 35, 36, 38-44, 46, 48, 49, 51-54, 56, 57], eight used a version of the EQ-5D [11,15,34,37,47,50,58,59], three used a version of AQoL instrument [21,22,57], one used the 15D [45] and one used the SF-6D [55].…”

“…Lopez-Bastida et al [47] reported that children with Type II spinal muscular atrophy tended to have lower mean proxy utility scores (− 0.01; ±0.35) than the combined average for all forms of spinal muscular atrophy (0.16; ± 0.44), however statistical analysis was not undertaken.…”