Base-case and probabilistic sensitivity analyses for rVWF vs pdVWF prophylaxis across all accepted WTP thresholds in the United States
| Prophylactic treatment . | Cost (95% credible interval), million USD . | Effectiveness (95% credible interval), QALY . | % Cost-effective at WTP $50 000/QALY . | % Cost-effective at WTP $100 000/QALY . | % Cost-effective at WTP $150 000/QALY . |
|---|---|---|---|---|---|
| rVWF | 17.0 (16.6-17.6) | 22.5 (21.1-23.9) | 100 | 100 | 100 |
| pdVWF | 18.9 (18.5-19.5) | 22.5 (21.1-23.9) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $1 880 000 ($1 560 000-$2 190 000) | |||||
| Scenario analysis: significant bleed reduction with rVWF | |||||
| rVWF | 16.9 (16.6-17.3) | 22.5 (21.1-23.9) | 100 | 100 | 100 |
| pdVWF | 18.9 (18.5-19.5) | 22.5 (21.1-23.9) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $2 020 000 ($1 350 000-$2 790 000) | |||||
| Scenario analysis: breakthrough bleed management with rFVIII | |||||
| rVWF | 17.0 (16.6-17.7) | 22.5 (21.1-23.9) | 100 | 100 | 100 |
| pdVWF | 18.9 (18.5-19.5) | 22.5 (21.1-23.9) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $1 840 000 ($1 520 000-$2 160 000) | |||||
| Scenario analysis: 5-ytime horizon | |||||
| rVWF | 3.3 (3.2-3.5) | 4.4 (4.1-4.7) | 100 | 100 | 100 |
| pdVWF | 3.7 (3.6-3.8) | 4.4 (4.1-4.7) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $370 000 ($310 000-$430 000) | |||||
| Prophylactic treatment . | Cost (95% credible interval), million USD . | Effectiveness (95% credible interval), QALY . | % Cost-effective at WTP $50 000/QALY . | % Cost-effective at WTP $100 000/QALY . | % Cost-effective at WTP $150 000/QALY . |
|---|---|---|---|---|---|
| rVWF | 17.0 (16.6-17.6) | 22.5 (21.1-23.9) | 100 | 100 | 100 |
| pdVWF | 18.9 (18.5-19.5) | 22.5 (21.1-23.9) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $1 880 000 ($1 560 000-$2 190 000) | |||||
| Scenario analysis: significant bleed reduction with rVWF | |||||
| rVWF | 16.9 (16.6-17.3) | 22.5 (21.1-23.9) | 100 | 100 | 100 |
| pdVWF | 18.9 (18.5-19.5) | 22.5 (21.1-23.9) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $2 020 000 ($1 350 000-$2 790 000) | |||||
| Scenario analysis: breakthrough bleed management with rFVIII | |||||
| rVWF | 17.0 (16.6-17.7) | 22.5 (21.1-23.9) | 100 | 100 | 100 |
| pdVWF | 18.9 (18.5-19.5) | 22.5 (21.1-23.9) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $1 840 000 ($1 520 000-$2 160 000) | |||||
| Scenario analysis: 5-ytime horizon | |||||
| rVWF | 3.3 (3.2-3.5) | 4.4 (4.1-4.7) | 100 | 100 | 100 |
| pdVWF | 3.7 (3.6-3.8) | 4.4 (4.1-4.7) | 0 | 0 | 0 |
| Incremental net monetary benefit at WTP $50 000-$150 000/QALY: $370 000 ($310 000-$430 000) | |||||
All point estimates rounded to maximum 3 significant digits.
QALY, quality-adjusted life year; rFVIII, recombinant factor VIII; USD, US dollar; WTP, willingness to pay.