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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 SMAArm 2: SMA NBS + Pre-symptomatic treatment for SMAArm 3: SMA NBS + Pre-symptomatic treatment for SMA with ≤ 3 SMN2 gene copies
Costs (2019 USD)
Screening costsNA$100,000$100,000
Reflex test costsNA$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   
QALYs269,987.67269,996.67269,996.09
Incremental results Arm 2 vs. Arm 1Arm 3 vs. Arm 1
Incremental costs$521,971-$142,303
Incremental QALYs9.008.42
Incremental costs per QALY gained$57,969Dominant
  1. NA not applicable, NBS newborn screening, QALYs Quality-Adjusted Life Years, SMA spinal muscular atrophy, SMN survival motor neuron, USD United States Dollar