Background
Methods
Survival
Quality of life
Costs
Cost-effectiveness
Model settings
Sensitivity analyses
Results
Patient population
Mean | Median | Range | |
---|---|---|---|
Age at first visit | 49.1 years | 50.0 years | 23.0–75.0 years |
Disease duration (since diagnosis) | 7.7 years | 4.3 years | 0.0–27.6 years |
Female | 48% | ||
Residual Enzyme activity (in fibroblasts) | 12.0% | 12.0% | 0.5–19.9% |
Wheelchair use | 31% | ||
Ventilation use | 27% | ||
Period at risk in ST survival analyses | 3.5 years | 3.5 years | 0.0–8.9 years |
Period at risk in ERT survival analyses | 3.4 years | 3.7 years | 0.2–8.4 years |
Survival
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] |
Quality of life
Costs and cost-effectiveness
Sensitivity analyses
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 |