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Table 3 Results one-way sensitivity analyses

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

  Incr. LY Incr. QALY Incr. Costs Cost/LY Cost/QALY
Scenario 1: No extrapolation of survival gains
Base Case 2.03 2.13 € 6,486,112 € 3,195,040 € 3,050,814
Structural uncertainty
 Survival: ERT as only covariate 2.15 2.08 € 5,780,738 € 2,692,018 € 2,772,920
 5 year time horizon 0.13 0.29 € 2,043,440 € 15,558,121 € 6,958,412
 15 year time horizon 1.19 1.31 € 4,681,908 € 3,944,770 € 3,567,548
Input values
 Medication costs 20% reduced 2.03 2.13 € 5,234,010 € 2,578,258 € 2,461,874
 Discount rates 0% 2.61 2.69 € 11,186,321 € 4,287,545 € 4,162,930
 No utility gain 2.03 1.26 € 6,486,112 € 3,195,040 € 5,138,186
 Utility gain of 0.10 2.03 3.03 € 6,486,112 € 3,195,040 € 2,139,947
 No difference in healthcare costs except for cost of ERT 2.03 2.13 € 6,418,842 € 3,161,903 € 3,019,173
 Survival: last value carried forward from year 8 onwards 1.98 2.08 € 6,387,051 € 3,231,592 € 3,073,535
Scenario 2: Extrapolated survival gains
Base Case 5.67 4.38 € 7,564,035 € 1,334,081 € 1,726,636
Structural uncertainty
 Survival: ERT as only covariate 5.44 4.14 € 6,749,881 € 1,241,847 € 1,629,079
 5 year time horizon 0.13 0.29 € 2,043,440 € 15,558,121 € 6,958,412
 15 year time horizon 1.59 1.58 € 4,836,654 € 3,047,385 € 3,053,857
Input values
 Medication costs 20% reduced 5.67 4.38 € 6,121,967 € 1,079,741 € 1,397,456
 Discount rates 0% 8.22 6.12 € 14,480,052 € 1,761,281 € 2,367,014
 No utility gain 5.67 3.34 € 7,564,035 € 1,334,081 € 2,265,117
 Utility gain of 0.10 5.67 5.47 € 7,564,035 € 1,334,081 € 1,382,324
 No difference in healthcare costs except for cost of ERT 5.67 4.38 € 7,390,957 € 1,303,555 € 1,687,127
 Survival: last value carried forward from year 8 onwards 5,65 4.36 € 7,478,037 € 1,323,777 € 1,716,538
  1. Base case results differ somewhat from Table 2 as sensitivity analyses were performed using deterministic analyses