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Table 4 Incremental cost-effectiveness ratios (dosage 40 mg/kg/week unless otherwise specified)

From: Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in classic-infantile patients with Pompe disease

 

Costs/QALY gained

Costs/life year gained

Base case analysis dosage 40 mg/kg/week

€ 1,043,868

€ 525,873

Registered dosage regimen (20 mg/kg/2 weeks)

€ 286,114

€ 144,137

Lower treatment costs (costs divided by 2)

€ 549,280

€ 276,713

Shorter time horizon (5 years)

€ 571,701

€ 92,634

Lower utility (0.49) in both cohorts

€ 1,304,835

€ 525,873

Higher utility (0.74) in both cohorts

€ 869,890

€ 525,873

Lower utility in ST-treated patients

€ 1,021,610

€ 525,873

Lognormal survival distribution

€ 1,050,595

€ 452,669

No costs incurred by ST-treated patients

€ 1,049,203

€ 528,560

Double cost incurred by ST-treated patients

€ 1,031,836

€ 520,387