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Table 2 Cost, effects and cost-effectiveness of ST and ERT (lifetime time horizon)

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

Scenario 1: No extrapolation of survival gains
Incremental and total effects ST ERT Difference Difference 95% CI
 Utilities 0.42 0.45 0.03 [0.02–0.05]
 Life expectancy 16.33 18.21 1.89 [0.67–2.62]
 QALYs 10.53 12.57 2.04 [1.30–2.57]
Incremental and total costs ST ERT Difference Difference 95% CI
 Total costs € 329,105 € 6,795,495 € 6,466,827 [€ 5,686,402-€ 7,340,316]
 Healthcare costs € 325,720 € 6,790,671 € 6,464,951 [€ 5,683,798-€ 7,342,186]
 ERT costs € 0 € 6,258,915 € 6,258,915 [€ 5,513,466-€ 7,019,921]
 Costs of drug administration € 0 € 157,457 € 157,457 [€ 95,399-€ 223,444]
 Other healthcare costs € 325,720 € 374,299 € 49,390 [€ -264,511-€ 301,652]
 Productivity costs € 3411 € 5662 € 2268 [€ -2916-€ 13,822]
Incremental cost-effectiveness ratios
 Cost / life year gained   € 3,417,713   [€ 2,237,739-€ 10,714,797]
 Cost / QALY gained   € 3,167,914   [€ 2,348,946-€ 5,485,622]
Scenario 2: Extrapolated survival gains
Incremental and total effects ST ERT Difference Difference 95% CI
 Utilities 0.42 0.45 0.03 [0.02–0.05]
 Life expectancy 16.42 21.84 5.44 [1.24–9.21]
 QALYs 10.60 14.85 4.26 [1.77–6.62]
Incremental and total costs ST ERT Difference Difference 95% CI
 Total costs € 324,967 € 7,879,226 € 7,554,844 [€ 6,885,851-€ 8,210,521]
 Healthcare costs € 321,558 € 7,874,627 € 7,553,917 [€ 6,844,436-€ 8,210,008]
 ERT costs € 0 € 7,206,219 € 7,206,219 [€ 6,684,091-€ 7,705,496]
 Costs of drug administration € 0 € 179,589 € 179,859 [€ 106,760-€ 257,239]
 Other healthcare costs € 321,558 € 488,819 € 168,109 [€ -172,810-€ 508,039]
 Productivity costs € 3435 € 5590 € 2173 [€ -2684-€ 12,621]
Incremental cost-effectiveness ratios
 Cost / life year gained   € 1,389,925   [€ 838,539-€ 5,317,415]
 Cost / QALY gained   € 1,774,390   [€ 1,164,826-€ 4,159,592]
  1. Discount rate effects 1.5%; Discount rate costs 4.0%