Table 3 Results one-way sensitivity analyses
| Â | 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 | |||||
âSurvival: ERT as only covariate | 2.15 | 2.08 | ⏠5,780,738 | ⏠2,692,018 | ⏠2,772,920 |
â5 year time horizon | 0.13 | 0.29 | ⏠2,043,440 | ⏠15,558,121 | ⏠6,958,412 |
â15 year time horizon | 1.19 | 1.31 | ⏠4,681,908 | ⏠3,944,770 | ⏠3,567,548 |
Input values | |||||
âMedication costs 20% reduced | 2.03 | 2.13 | ⏠5,234,010 | ⏠2,578,258 | ⏠2,461,874 |
âDiscount rates 0% | 2.61 | 2.69 | ⏠11,186,321 | ⏠4,287,545 | ⏠4,162,930 |
âNo utility gain | 2.03 | 1.26 | ⏠6,486,112 | ⏠3,195,040 | ⏠5,138,186 |
âUtility gain of 0.10 | 2.03 | 3.03 | ⏠6,486,112 | ⏠3,195,040 | ⏠2,139,947 |
âNo difference in healthcare costs except for cost of ERT | 2.03 | 2.13 | ⏠6,418,842 | ⏠3,161,903 | ⏠3,019,173 |
âSurvival: 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 | |||||
âSurvival: ERT as only covariate | 5.44 | 4.14 | ⏠6,749,881 | ⏠1,241,847 | ⏠1,629,079 |
â5 year time horizon | 0.13 | 0.29 | ⏠2,043,440 | ⏠15,558,121 | ⏠6,958,412 |
â15 year time horizon | 1.59 | 1.58 | ⏠4,836,654 | ⏠3,047,385 | ⏠3,053,857 |
Input values | |||||
âMedication costs 20% reduced | 5.67 | 4.38 | ⏠6,121,967 | ⏠1,079,741 | ⏠1,397,456 |
âDiscount rates 0% | 8.22 | 6.12 | ⏠14,480,052 | ⏠1,761,281 | ⏠2,367,014 |
âNo utility gain | 5.67 | 3.34 | ⏠7,564,035 | ⏠1,334,081 | ⏠2,265,117 |
âUtility gain of 0.10 | 5.67 | 5.47 | ⏠7,564,035 | ⏠1,334,081 | ⏠1,382,324 |
âNo difference in healthcare costs except for cost of ERT | 5.67 | 4.38 | ⏠7,390,957 | ⏠1,303,555 | ⏠1,687,127 |
âSurvival: last value carried forward from year 8 onwards | 5,65 | 4.36 | ⏠7,478,037 | ⏠1,323,777 | ⏠1,716,538 |