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Table 1 Overview of literature on cost of SMA

From: Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

References Country
Year
Sample size Population age Type of study Perspective Type of cost Year of costing Funding
Armstrong et al. [24] USA
2016
239:
 45 < 1 year
 194 > 1 year
7.5+/− 6.4 Cross-sectional, retrospective, and prospective Healthcare costs Direct healthcare 2003–2012 Conducted by Biogen
Chambers et al. [31] Australia
2020
40:
 4 SMA1
 26 SMA2
 10 SMA3
SMA1: 2.7 (1–5)
SMA2: 9.8 (2–22)
SMA3: 6.9 (1–12)
Cross-sectional
retrospective
Societal costs Direct healthcare
Direct non-healthcare
Indirect
2016–2017 Funded by the Motor Neurone Diseases Research institute of Australia Beryl Bayley
Darba et al. [27] Spain
2020
396 SMA1, 2 3 and 4   Cross-sectional
retrospective
Healthcare costs Direct healthcare 2014–2016 No
Droege et al. [29] USA
2019
6526:
 349 SMA1
 45 SMA1 treated with nusinersen
 5728 SMA2, 3, 4
 404 SMA2, 3, 4 treated with nusinersen
SMA1: 9.2 months
SMA1 nusinersen: 12.2 months
SMA others: 30.9 years
SMA others nusinersen: 14.8 years
Retrospective Healthcare costs Direct healthcare 09/2016–08/2018 Conducted by Avexis
Klug et al. (30) Germany
2016
189:
 12 SMA1
 73 SMA2
 104 SMA3
 < 1 to 73 Cross-sectional
retrospective
Healthcare and societal costs Direct healthcare
Direct non-healthcare
Indirect
2013 Grant of the Friedrich-Baur-GmbH m’
Lee et al. (25) USA
2019
229 severe SMA (< 1 year)   Cross-sectional
retrospective
Healthcare costs Direct healthcare 2005–2013 No
Lewin Group (23) USA
2012
745:
 14 early onset SMA
 731 SMA other (3–4)
 < 1 to 65 Cross-sectional
retrospective
Healthcare and societal costs Direct healthcare
Direct non-healthcare
Indirect
2008 Conducted by Muscular Dystrophy Association
Lopez-Bastida et al. [61] Spain
2017
81:
 8 SMA1
 60 SMA2
 13 SMA3
7.22 Cross-sectional
retrospective
Healthcare and societal costs Direct healthcare
Direct non-healthcare
2015 Supported by Biogen
Peña-Longobardo et al. [28] France, German, UK
2020
86:
 23 SMA1
 45 SMA2
 18 SMA 3
6.9 Cross-sectional
prospective
Societal costs Direct healthcare
Direct non-healthcare
2015 Supported by Biogen