TABLE I.
Economic model inputs for efficacy and utilities
| Input parameter | Base value | Probability distribution [mean (sd)] | Source |
|---|---|---|---|
| Efficacy | Individual patient data from the aza-001 trial | ||
| Modeled median os (months)a | |||
| Preselected for bsc | |||
| Azacitidine | 25.52 | Normal | |
| bsc | 14.69 | Normal | |
| Preselected for ldc | |||
| Azacitidine | 25.48 | Normal | |
| ldc | 16.38 | Normal | |
| Preselected for sdc | |||
| Azacitidine | 21.98 | Normal | |
| sdc | 16.05 | Normal | |
| Transformation to aml (>30% blasts) | 37.7% | Fixed value | |
| Utilities | |||
| Azacitidine | Celgene utility study, calgb 9221 study Kornblith et al.7 | ||
| Day 0 | 0.67 | Beta: 0.67 (0.22) | |
| Day 50 | 0.70 | Beta; 0.70 (0.20) | |
| Day 106 | 0.74 | Beta; 0.74 (0.20) | |
| Day 182 | 0.80 | Beta; 0.80 (0.21) | |
| Day 183 onward | 0.80 | Assumption | Data after day 182 were not available, assumed to be the same as day 182 |
| Best supportive care | Celgene utility study, calgb 9221 study Kornblith et al.7 | ||
| Day 0 | 0.67 | Beta; 0.67 (0.22) | |
| Day 50 | 0.69 | Beta; 0.69 (0.20) | |
| Day 106 | 0.68 | Beta; 0.68 (0.22) | |
| Day 182 | 0.72 | Beta; 0.72 (0.22) | |
| Day 183 onward | 0.80 | Assumption | Data after day 182 were not available, assumed to be the same as day 182 |
| Low-dose chemotherapy | Celgene utility study; Sekeres et al.8 | ||
| Day 0 | 0.67 | Beta; 0.67 (0.08) | |
| Day 14 | 0.70 | Beta; 0.70 (0.09) | |
| Day 42 | 0.71 | Beta; 0.71(0.15) | |
| Day 70 | 0.72 | Beta; 0.72 (0.13) | |
| Day 98 | 0.70 | Beta; 0.70 (0.06) | |
| Day 182 | 0.85 | Beta; 0.85 (0.08) | |
| Day 365 | 0.67 | Beta; 0.67 (0.22) | |
| Day 366 onward | 0.67 | Assumption | Data after day 365 were not available, assumed to be the same as day 365 |
| Standard-dose chemotherapy | Celgene utility study, Sekeres et al.8 | ||
| Day 0 | 0.66 | Beta; 0.66 (0.13) | |
| Day 14 | 0.61 | Beta; 0.61 (0.10) | |
| Day 42 | 0.66 | Beta; 0.66 (0.10) | |
| Day 70 | 0.69 | Beta; 0.69 (0.12) | |
| Day 98 | 0.72 | Beta; 0.72 (0.16) | |
| Day 182 | 0.74 | Beta; 0.74 (0.18) | |
| Day 365 | 0.83 | Beta; 0.83 (0.10) | |
| Day 366 onward | 0.83 | Assumption | Data after day 365 were not available, assumed to be the same as day 365 |
| aml (>30% blasts) | 0.67 | Beta; 0.67 (0.22) | Assumption |
| Discount rate | |||
| For costs and outcomes | 5% | Fixed value |
a
Weibull extrapolation: converted from 5-week cycles.
sd = standard deviation; os = overall survival; bsc = best supportive care; ldc = low-dose chemotherapy; sdc = standard-dose chemotherapy; aml = acute myeloid leukemia; calgb = Cancer and Leukemia Group B.