Base-case estimates and ranges used in sensitivity analysis
| Variables . | Base-case estimate . | Range . | References . | Notes . |
|---|---|---|---|---|
| Clinical probabilities | ||||
| Bleeding events | ||||
| Prophylaxis | 0.3/mo | 2-4/y | 23 | |
| Hospitalization | 0.0175/mo | 0.01-0.02/mo | 25 | |
| Joint complications | ||||
| Joint bleed | 0.39/mo | 0.3-0.5/mo | 26 | |
| Hospitalization | 0.10/event | 0.05-0.15/mo | Range estimated | |
| Orthopedic procedure | 2/lifetime (0.0022/mo) | 1-3/lifetime | 27 | |
| Gene therapy | ||||
| Success | 0.90 | 0.8-1 | Range estimated | |
| Complications | 0.01 | 0.0005-0.002 | Range estimated | |
| Death | 0.00013/mo | 0.0001-0.0005/mo | 21 | Mean risk for death, 30-40-y-olds (CDC) |
| Time courses | ||||
| Bleeding event | 2 d | 2-4 d | 7 | |
| Hospitalization | 4 d | 2-6 d | 32 | Median hemophilia admission (HCUP) |
| Joint damage | 14 d | 7-21 d | ||
| Gene therapy complication | 4 d | — | ||
| Quality-of-life measures (utilities) | ||||
| Prophylaxis | 0.93 | 0.87-0.93 | 22, 28 | |
| Hospitalization | 0.66 | 0.5-0.7 | Based on bleed utility | |
| Bleed | 0.66 | 0.66-0.8 | 33 | |
| Joint damage | 0.64 | — | 30 | |
| Gene therapy | 1 | 0.9-1 | Assumed utility | |
| Gene therapy complication | 0.8 | 0.7-0.9 | Assumed utility | |
| Costs, US$ | ||||
| Prophylaxis | 37 759 | — | 7, 31 | Based on 88.7-kg male, 33 IU/kg 3 times weekly |
| Bleeding event | 8 870 | 4 435-17 740 | 7 | Based on 50 IU/kg/d for bleed duration |
| Hospitalization for bleeding event | 48 603 | 33 582-63 678 | 7, 32 | Average Medicare reimbursement (HCUP) for hospitalization plus factor at 50 IU/kg/d |
| Joint damage | 61 | 40-80 | 33 | |
| Orthopedic surgery (knee replacement) | 137 461 | 110 000-138 000 | 32 | Average Medicare reimbursement (HCUP) for knee replacement plus factor |
| Gene therapy | 850 000 | 10 000-2 100 000 | 20 | |
| Gene therapy failure | 800 | 500-1 000 | 20 |
| Variables . | Base-case estimate . | Range . | References . | Notes . |
|---|---|---|---|---|
| Clinical probabilities | ||||
| Bleeding events | ||||
| Prophylaxis | 0.3/mo | 2-4/y | 23 | |
| Hospitalization | 0.0175/mo | 0.01-0.02/mo | 25 | |
| Joint complications | ||||
| Joint bleed | 0.39/mo | 0.3-0.5/mo | 26 | |
| Hospitalization | 0.10/event | 0.05-0.15/mo | Range estimated | |
| Orthopedic procedure | 2/lifetime (0.0022/mo) | 1-3/lifetime | 27 | |
| Gene therapy | ||||
| Success | 0.90 | 0.8-1 | Range estimated | |
| Complications | 0.01 | 0.0005-0.002 | Range estimated | |
| Death | 0.00013/mo | 0.0001-0.0005/mo | 21 | Mean risk for death, 30-40-y-olds (CDC) |
| Time courses | ||||
| Bleeding event | 2 d | 2-4 d | 7 | |
| Hospitalization | 4 d | 2-6 d | 32 | Median hemophilia admission (HCUP) |
| Joint damage | 14 d | 7-21 d | ||
| Gene therapy complication | 4 d | — | ||
| Quality-of-life measures (utilities) | ||||
| Prophylaxis | 0.93 | 0.87-0.93 | 22, 28 | |
| Hospitalization | 0.66 | 0.5-0.7 | Based on bleed utility | |
| Bleed | 0.66 | 0.66-0.8 | 33 | |
| Joint damage | 0.64 | — | 30 | |
| Gene therapy | 1 | 0.9-1 | Assumed utility | |
| Gene therapy complication | 0.8 | 0.7-0.9 | Assumed utility | |
| Costs, US$ | ||||
| Prophylaxis | 37 759 | — | 7, 31 | Based on 88.7-kg male, 33 IU/kg 3 times weekly |
| Bleeding event | 8 870 | 4 435-17 740 | 7 | Based on 50 IU/kg/d for bleed duration |
| Hospitalization for bleeding event | 48 603 | 33 582-63 678 | 7, 32 | Average Medicare reimbursement (HCUP) for hospitalization plus factor at 50 IU/kg/d |
| Joint damage | 61 | 40-80 | 33 | |
| Orthopedic surgery (knee replacement) | 137 461 | 110 000-138 000 | 32 | Average Medicare reimbursement (HCUP) for knee replacement plus factor |
| Gene therapy | 850 000 | 10 000-2 100 000 | 20 | |
| Gene therapy failure | 800 | 500-1 000 | 20 |
The base-case estimates represent the best estimate for each value. Unless otherwise noted, ranges represent 95% confidence intervals.
CDC, Centers for Disease Control and Prevention.