Table 2 Base-case results (per 10,000 newborns)
From: 10th European Conference on Rare Diseases & Orphan Products (ECRD 2020)
|  | Arm 1: No SMA screening + Symptomatic treatment for SMA | Arm 2: SMA NBS + Pre-symptomatic treatment for SMA | Arm 3: SMA NBS + Pre-symptomatic treatment for SMA with ≤ 3 SMN2 gene copies |
|---|---|---|---|
Costs (2019 USD) | |||
Screening costs | NA | $100,000 | $100,000 |
Reflex test costs | NA | $31 | $31 |
Treatment costs | $2,628,116 | $3,050,056 | $2,385,782 |
Total costs | $2,628,116 | $3,150,087 | $2,485,813 |
Effectiveness | Â | Â | Â |
QALYs | 269,987.67 | 269,996.67 | 269,996.09 |
Incremental results | Â | Arm 2 vs. Arm 1 | Arm 3 vs. Arm 1 |
Incremental costs | – | $521,971 | -$142,303 |
Incremental QALYs | – | 9.00 | 8.42 |
Incremental costs per QALY gained | – | $57,969 | Dominant |