Table 2. Results of the Base Case Analysis and Scenario Analyses Varying the Reduction in the Long-term Treatment Effect After Year 3.
| Variable | Cost Comparison, $a | ||
|---|---|---|---|
| Voretigene Neparvovec-rzyl | SC | Incremental Value (Voretigene Neparvovec-rzyl − SC) | |
| Base Case, Lifetime Treatment Effect | |||
| Direct medical costs, $ | 301 794 | 406 404 | − 104 610 |
| Drug | 854 535 | 0 | 854 535 |
| Indirect | 1 063 739 | 2 373 702 | −1 309 963 |
| Total | 2 220 069 | 2 780 106 | −560 038 |
| QALYs | 18.1 | 8.6 | 9.4 |
| ICER | |||
| With indirect costs | NA | NA | −59 458 Voretigene neparvovec-rzyl lower costs and higher QALYs |
| Without indirect costs | NA | NA | 79 618 |
| Voretigene Neparvovec-rzyl With 5% Reduction in Long-term Treatment Effect After Year 3 | |||
| Direct medical costs, $ | 305 780 | 406 404 | −100 625 |
| Drug | 854 535 | 0 | 854 535 |
| Indirect | 1 113 916 | 2 373 702 | −1 259 786 |
| Total | 2 274 232 | 2 780 106 | − 505 875 |
| QALYs | 17.7 | 8.6 | 9.1 |
| ICER | |||
| With indirect costs | NA | NA | − 55 869 |
| Without indirect costs | NA | NA | 83 262 |
| Voretigene Neparvovec-rzyl With 10% Reduction in Long-term Treatment Effect After Year 3 | |||
| Direct medical costs, $ | 309 763 | 406 404 | −96 641 |
| Drug | 854 535 | 0 | 854 535 |
| Indirect | 1 164 064 | 2 373 702 | − 1 209 638 |
| Total | 2 328 362 | 2 780 106 | −451 744 |
| QALYs | 17.3 | 8.6 | 8.7 |
| ICER | |||
| With indirect costs | NA | NA | −51 981 |
| Without indirect costs | NA | NA | 87 209 |
| Voretigene Neparvovec-rzyl With 50% Reduction in Long-term Treatment Effect After Year 3 | |||
| Direct medical costs, $ | 341 550 | 406 404 | −64 855 |
| Drug | 854 535 | 0 | 854 535 |
| Indirect | 1 564 180 | 2 373 702 | −809 522 |
| Total | 2 760 265 | 2 780 106 | −19 842 |
| QALYs | 14.4 | 8.6 | 5.8 |
| ICER | |||
| With indirect costs | −3429 | ||
| Without indirect costs | 136 452 | ||
| Voretigene Neparvovec-rzyl With 100% Reduction in Long-term Treatment Effect After Year 3 | |||
| Direct medical costs, $ | 381 087 | 406 404 | −25 318 |
| Drug | 854 535 | 0 | 854 535 |
| Indirect | 2 061 709 | 2 373 702 | −311 993 |
| Total | 3 297 331 | 2 780 106 | 517 225 |
| QALYs | 10.8 | 8.6 | 2.2 |
| ICER | |||
| With indirect costs | 237 140 | ||
| Without indirect costs | 380 185 | ||
Abbreviations: ICER, incremental cost-effectiveness ratio; QALY, quality-adjusted life-year; SC, standard care; TE, treatment effect; VI, visual impairment.
a
Values are discounted.