Base-case and scenario analysis model input parameters and distributions
| . | Base case . | Distributions . | References . |
|---|---|---|---|
| Clinical parameters | |||
| Age, y | 51 | 13 | |
| Eculizumab use in SOC, % | 66 | 13 | |
| Transfusion avoidance in patients treated with iptacopan, % | 94.8 | PERT (88.1-100.0) | 13 |
| Transfusion avoidance in patients treated with SOC, % | 25.9 | PERT (11.6-42.4) | 13 |
| Monthly probability of BTH in iptacopan | 0.0083 | PERT (0-0.0247) | 13 |
| Monthly probability of BTH in SOC | 0.0567 | PERT (0.0247-0.132) | 13 |
| Number of RBC units in transfusion-dependent patients in 6 mo | 4 | Gamma (0.89, 7.62) | 13 |
| Number of RBC units for BTH treatment | 2 | Gamma (4, 0.5) | 13 |
| Annual DHTR incidence, per product | 0.0000526 | PERT (0.0000421-0.0000632) | 20 |
| Annual TRALI incidence, per product | 0.0000175 | PERT (0.0000140-0.0000211) | 20 |
| Annual TACO incidence, per product | 0.000111 | PERT (0.0000889-0.000133) | 20 |
| DHTR mortality | 0.038 | PERT (0-0.064) | 21 |
| TRALI mortality | 0.075 | PERT (0.06-0.09) | 22 |
| TACO mortality | 0.084 | PERT (0.067-0.10) | 23 |
| Utilities | |||
| Transfusion independent (Hb ≥ 10.5 g/dL) | 0.808 | PERT (0.646-0.970) | 17 |
| Transfusion dependent | 0.695 | PERT (0.556-0.834) | 17 |
| Transfusion independent (PRINCE) | 0.869 | PERT (0.695-1) | 25 |
| Transfusion dependent (PRINCE) | 0.818 | PERT (0.556-0.799) | 25 |
| General population utility (female-to-male sex ratio (%), 69:31 | Age dependent | 26 | |
| Moderate anemia disutility | 0.052 | PERT (0.034-0.076) | 27 |
| Utility increment by oral therapy vs injection | 0.01 | PERT (0.008-0.012) | 28,29 |
| DHTR disutility | 0.4 | PERT (0.32-0.48) | 27 |
| TRALI disutility | 0.4 | PERT (0.32-0.48) | 30 |
| TACO disutility | 0.4 | PERT (0.32-0.48) | Assumption |
| BTH disutility | 0.4 | PERT (0.32-0.48) | 31 |
| Costs | |||
| US cost ($) | |||
| Iptacopan, per mg | 3.34 | 32 | |
| Eculizumab, per mg | 22.70 | 33 | |
| Ravulizumab, per mg | 22.27 | 33 | |
| Iptacopan, per mg (AWP) | 4.52 | 34 | |
| Eculizumab, per mg (AWP) | 26.09 | 34 | |
| Ravulizumab, per mg (AWP) | 25.62 | 34 | |
| Cost of administration | 160.63 | Gamma (100, 1.61) | 35 |
| RBC unit cost | 196.20 | Gamma (100, 1.96) | 36 |
| Transfusion administration | 1 240 | Gamma (100, 12.4) | 36 |
| Cost of DHTR | 1 537 | Gamma (100, 15.4) | 37 |
| Cost of TRALI | 9 957 | Gamma (100, 99.6) | 37 |
| Cost of TACO | 4 830 | Gamma (100, 48.3) | 37 |
| All-cause direct medical cost due to transfusion dependent (PPPY) | 252 010 | Gamma (100, 2520) | 38 |
| All-cause medical related absenteeism cost due to transfusion dependent (PPPY) | 4 935 | Gamma (100, 49.4) | 38 |
| Mean hourly wage (all occupation) | 29.76 | Gamma (100, 0.30) | 39 |
| Mean hourly wage (registered nurse) | 42.80 | Gamma (100, 0.43) | 39 |
| Japanese cost (¥) | |||
| Eculizumab (300 mg/30 mL) | 619 834 | 40 | |
| Ravulizumab (300 mg/3 mL) | 699 570 | 40 | |
| Eculizumab administration cost | 7 856 | 41 | |
| Ravulizumab administration cost | 11 761 | 41 | |
| RBC unit cost | 9 067 | 40 | |
| Transfusion administration | 4 500 | 42 | |
| Absenteeism cost | 387 345 | Gamma (100, 3 873) | 38 |
| Mean hourly wage (all employees) | 2 336 | Gamma (100, 23.4) | 43 |
| Mean hourly wage (nurse) | 2 633 | Gamma (100, 26.3) | 43 |
| Brazilian cost (R$) | |||
| Eculizumab (300 mg/30 mL) | 19 016.68 | 44 | |
| Ravulizumab (300 mg/3 mL) | 20 540.12 | 44 | |
| Administration cost | 0.63 | 18 | |
| RBC unit cost | 148.80 | 45 | |
| Transfusion administration | 8.09 | 18,46 | |
| Absenteeism cost | 4635 | Gamma (100, 46.4) | 38 |
| Mean hourly wage (all employees) | 27.95 | Gamma (100, 0.28) | 47 |
| Mean hourly wage (nurse) | 33.50 | Gamma (100, 0.34) | 48,49 |
| . | Base case . | Distributions . | References . |
|---|---|---|---|
| Clinical parameters | |||
| Age, y | 51 | 13 | |
| Eculizumab use in SOC, % | 66 | 13 | |
| Transfusion avoidance in patients treated with iptacopan, % | 94.8 | PERT (88.1-100.0) | 13 |
| Transfusion avoidance in patients treated with SOC, % | 25.9 | PERT (11.6-42.4) | 13 |
| Monthly probability of BTH in iptacopan | 0.0083 | PERT (0-0.0247) | 13 |
| Monthly probability of BTH in SOC | 0.0567 | PERT (0.0247-0.132) | 13 |
| Number of RBC units in transfusion-dependent patients in 6 mo | 4 | Gamma (0.89, 7.62) | 13 |
| Number of RBC units for BTH treatment | 2 | Gamma (4, 0.5) | 13 |
| Annual DHTR incidence, per product | 0.0000526 | PERT (0.0000421-0.0000632) | 20 |
| Annual TRALI incidence, per product | 0.0000175 | PERT (0.0000140-0.0000211) | 20 |
| Annual TACO incidence, per product | 0.000111 | PERT (0.0000889-0.000133) | 20 |
| DHTR mortality | 0.038 | PERT (0-0.064) | 21 |
| TRALI mortality | 0.075 | PERT (0.06-0.09) | 22 |
| TACO mortality | 0.084 | PERT (0.067-0.10) | 23 |
| Utilities | |||
| Transfusion independent (Hb ≥ 10.5 g/dL) | 0.808 | PERT (0.646-0.970) | 17 |
| Transfusion dependent | 0.695 | PERT (0.556-0.834) | 17 |
| Transfusion independent (PRINCE) | 0.869 | PERT (0.695-1) | 25 |
| Transfusion dependent (PRINCE) | 0.818 | PERT (0.556-0.799) | 25 |
| General population utility (female-to-male sex ratio (%), 69:31 | Age dependent | 26 | |
| Moderate anemia disutility | 0.052 | PERT (0.034-0.076) | 27 |
| Utility increment by oral therapy vs injection | 0.01 | PERT (0.008-0.012) | 28,29 |
| DHTR disutility | 0.4 | PERT (0.32-0.48) | 27 |
| TRALI disutility | 0.4 | PERT (0.32-0.48) | 30 |
| TACO disutility | 0.4 | PERT (0.32-0.48) | Assumption |
| BTH disutility | 0.4 | PERT (0.32-0.48) | 31 |
| Costs | |||
| US cost ($) | |||
| Iptacopan, per mg | 3.34 | 32 | |
| Eculizumab, per mg | 22.70 | 33 | |
| Ravulizumab, per mg | 22.27 | 33 | |
| Iptacopan, per mg (AWP) | 4.52 | 34 | |
| Eculizumab, per mg (AWP) | 26.09 | 34 | |
| Ravulizumab, per mg (AWP) | 25.62 | 34 | |
| Cost of administration | 160.63 | Gamma (100, 1.61) | 35 |
| RBC unit cost | 196.20 | Gamma (100, 1.96) | 36 |
| Transfusion administration | 1 240 | Gamma (100, 12.4) | 36 |
| Cost of DHTR | 1 537 | Gamma (100, 15.4) | 37 |
| Cost of TRALI | 9 957 | Gamma (100, 99.6) | 37 |
| Cost of TACO | 4 830 | Gamma (100, 48.3) | 37 |
| All-cause direct medical cost due to transfusion dependent (PPPY) | 252 010 | Gamma (100, 2520) | 38 |
| All-cause medical related absenteeism cost due to transfusion dependent (PPPY) | 4 935 | Gamma (100, 49.4) | 38 |
| Mean hourly wage (all occupation) | 29.76 | Gamma (100, 0.30) | 39 |
| Mean hourly wage (registered nurse) | 42.80 | Gamma (100, 0.43) | 39 |
| Japanese cost (¥) | |||
| Eculizumab (300 mg/30 mL) | 619 834 | 40 | |
| Ravulizumab (300 mg/3 mL) | 699 570 | 40 | |
| Eculizumab administration cost | 7 856 | 41 | |
| Ravulizumab administration cost | 11 761 | 41 | |
| RBC unit cost | 9 067 | 40 | |
| Transfusion administration | 4 500 | 42 | |
| Absenteeism cost | 387 345 | Gamma (100, 3 873) | 38 |
| Mean hourly wage (all employees) | 2 336 | Gamma (100, 23.4) | 43 |
| Mean hourly wage (nurse) | 2 633 | Gamma (100, 26.3) | 43 |
| Brazilian cost (R$) | |||
| Eculizumab (300 mg/30 mL) | 19 016.68 | 44 | |
| Ravulizumab (300 mg/3 mL) | 20 540.12 | 44 | |
| Administration cost | 0.63 | 18 | |
| RBC unit cost | 148.80 | 45 | |
| Transfusion administration | 8.09 | 18,46 | |
| Absenteeism cost | 4635 | Gamma (100, 46.4) | 38 |
| Mean hourly wage (all employees) | 27.95 | Gamma (100, 0.28) | 47 |
| Mean hourly wage (nurse) | 33.50 | Gamma (100, 0.34) | 48,49 |
AWP, average wholesale price; DHTR, delayed hemolytic transfusion reaction; Hb, hemoglobin; PERT, project evaluation and review techniques; PPPY, per person per year; RBC, red blood cell; TACO, transfusion-associated circulatory overload; TRALI, transfusion-related acute lung injury.