Table 1 Overview of literature on cost of SMA
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: Ā 45ā<ā1Ā year Ā 194ā>ā1Ā year | 7.5+/āĀ 6.4 | Cross-sectional, retrospective, and prospective | Healthcare costs | Direct healthcare | 2003ā2012 | Conducted by Biogen |
Chambers et al. [31] | Australia 2020 | 40: Ā 4 SMA1 Ā 26 SMA2 Ā 10 SMA3 | SMA1: 2.7 (1ā5) SMA2: 9.8 (2ā22) SMA3: 6.9 (1ā12) | Cross-sectional retrospective | Societal costs | Direct healthcare Direct non-healthcare Indirect | 2016ā2017 | Funded by the Motor Neurone Diseases Research institute of Australia Beryl Bayley |
Darba et al. [27] | Spain 2020 | 396 SMA1, 2 3 and 4 | Ā | Cross-sectional retrospective | Healthcare costs | Direct healthcare | 2014ā2016 | No |
Droege et al. [29] | USA 2019 | 6526: Ā 349 SMA1 Ā 45 SMA1 treated with nusinersen Ā 5728 SMA2, 3, 4 Ā 404 SMA2, 3, 4 treated with nusinersen | SMA1: 9.2Ā months SMA1 nusinersen: 12.2Ā months SMA others: 30.9Ā years SMA others nusinersen: 14.8Ā years | Retrospective | Healthcare costs | Direct healthcare | 09/2016ā08/2018 | Conducted by Avexis |
Klug et al. (30) | Germany 2016 | 189: Ā 12 SMA1 Ā 73 SMA2 Ā 104 SMA3 | ā<ā1 to 73 | Cross-sectional retrospective | Healthcare and societal costs | Direct healthcare Direct non-healthcare Indirect | 2013 | Grant of the Friedrich-Baur-GmbH mā |
Lee et al. (25) | USA 2019 | 229 severe SMA (<ā1Ā year) | Ā | Cross-sectional retrospective | Healthcare costs | Direct healthcare | 2005ā2013 | No |
Lewin Group (23) | USA 2012 | 745: Ā 14 early onset SMA Ā 731 SMA other (3ā4) | ā<ā1 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: Ā 8 SMA1 Ā 60 SMA2 Ā 13 SMA3 | 7.22 | Cross-sectional retrospective | Healthcare and societal costs | Direct healthcare Direct non-healthcare | 2015 | Supported by Biogen |
PeƱa-Longobardo et al. [28] | France, German, UK 2020 | 86: Ā 23 SMA1 Ā 45 SMA2 Ā 18 SMA 3 | 6.9 | Cross-sectional prospective | Societal costs | Direct healthcare Direct non-healthcare | 2015 | Supported by Biogen |