Table 2 Overview of economic evaluation studies of SMA
References | Country | Perspective | Time horizon | Method | Outcomes used | Industry funding |
|---|---|---|---|---|---|---|
CADTH [34] | Canada 2018 | Healthcare payer | SMA1: 25 years SMA2: 50 years SMA3: 80 years | Three Markov models: for SMA1, for SMA2, for SMA3 | Life years QALY | No, commissioned by health authorities |
ICER [36] | England 2018 | Healthcare payer and societal perspective | Two scenarios: 5 years 10 years | Three Markov models: for SMA1, for SMA2 and SMA3, for pre-symptomatic SMA | QALY | No, commissioned by health authorities |
Jalali [32] | USA 2020 | Societal perspective | 30 months | Four Markov models: for untreated patients SMA1, for treated SMA1 identified by symptoms, for untreated patients identified by newborn screening, for nusinersen-treated patients identified by newborn screening | Life Years QALY | No |
Malone et al. [33] | USA 2019 | Healthcare payer | Lifetime horizon | Markov model comparing nusinersen and Onasemnogene abeparvovec for SMA1 | QALY | Avexis |
National Center for Pharmaco-economics [37] | Ireland 2017 | Societal perspective | Lifetime horizon | Two separate Markov models: for early-onset SMA, for later-onset SMA | QALY | No, commissioned by health authorities |
Zuluaga-Sanchez et al. [35] | Sweden 2018 | Societal and payer perspective | SMA1: 40 years SMA2: 80 years | Markov model: incremental cost QALY gained and overall survival. Two models: for early-onset SMA, for later-onset SMA | QALY | Biogen |