Table 4.
Scenario results
| Scenario category | Scenario parameters a | Third-party payer perspective | Societal perspective | ||||
|---|---|---|---|---|---|---|---|
| Incremental QALYs | Incremental costs | $/QALY gained (diff. versus base case) | Incremental QALYs | Incremental costs | $/QALY gained (diff. versus base case) | ||
| Base case | 10.20 | $1,952,808 | $191,519 | 11.39 | $1,412,393 | $124,051 | |
| Lovo-cel target population | Age: 12–17 years only b | 12.73 | $1,403,433 | $110,244 (−42.4%) | 14.55 | $705,202 | $48,460 (−60.9%) |
| Age: 18–30 years only b | 10.26 | $1,861,801 | $181,466 (−5.2%) | 11.44 | $1,320,751 | $115,437 (−6.9%) | |
| Age: > 30 years only b | 8.20 | $2,604,663 | $317,739 (+65.9%) | 8.95 | $2,179,382 | $243,405 (+96.2%) | |
| Prior VOE criteria: any history of VOEs | 8.60 | $2,468,155 | $286,896 (+49.8%) | 10.16 | $1,943,252 | $191,320 (+54.2%) | |
| Lovo-cel VOE and total Hb efficacy | VOE-CR: 96.5% of patients; sVOE-CR: 99.2% of patients | 10.92 | $1,921,783 | $176,064 (−8.1%) | 12.29 | $1,326,813 | $107,938 (−13.0%) |
| VOE-CR: 71.0% of patients; sVOE-CR: 79.2% of patients | 8.70 | $2,011,648 | $231,144 (+20.7%) | 9.60 | $1,572,267 | $163,826 (+32.1%) | |
| Partial VOE reduction: excluded | 10.08 | $2,014,516 | $199,907 (+4.4%) | 11.28 | $1,474,645 | $130,762 (+5.4%) | |
| Total Hb change from baseline: 4.10 g/dL (SD, 1.28) | 10.32 | $1,886,663 | $182,787 (−4.6%) | 11.51 | $1,344,109 | $116,778 (−5.9%) | |
| Total Hb change from baseline: 2.86 g/dL (SD, 1.70) | 10.00 | $2,045,616 | $204,517 (+6.8%) | 11.19 | $1,508,562 | $134,774 (+8.6%) | |
| Lovo-cel HRQOL impacts | Utility gain: 0.146 | 11.39 | $1,952,808 | $171,485 (−10.5%) | 12.58 | $1,412,393 | $112,301 (−9.5%) |
| Utility gain: 0.056 | 9.54 | $1,952,808 | $204,715 (+6.9%) | 10.73 | $1,412,393 | $131,651 (+6.1%) | |
| Partial loss of lovo-cel effect | 10% of patients lose 50% of VOE and Hb effects after 5 years | 9.45 | $1,978,202 | $209,276 (+9.3%) | 10.55 | $1,471,792 | $139,481 (+12.4%) |
| VOE-CR impact assumptions for lovo-cel | VOE-CR impact: 90%/80% reduction for ages 18–30/> 30 years | 10.72 | $1,961,672 | $182,992 (−4.5%) | 11.90 | $1,408,479 | $118,404 (−4.6%) |
| VOE-CR impact: 80%/60% reduction for ages 18–30/> 30 years | 9.73 | $1,944,138 | $199,708 (+4.3%) | 10.94 | $1,416,011 | $129,461 (+4.4%) | |
| Lovo-cel drug product price | 23.1% rebate on lovo-cel acquisition price | 10.20 | $1,236,708 | $121,289 (−36.7%) | 11.39 | $696,293 | $61,156 (−50.7%) |
| Event and complication costs and utilities | 25% increase in direct costs | 10.20 | $1,662,701 | $163,067 (−14.9%) | 11.39 | $1,122,285 | $98,571 (−20.5%) |
| 25% decrease in direct costs | 10.20 | $2,242,916 | $219,971 (+14.9%) | 11.39 | $1,702,500 | $149,531 (+20.5%) | |
| 25% increase in disutilities | 10.45 | $1,952,808 | $186,959 (−2.4%) | 11.63 | $1,412,393 | $121,399 (−2.1%) | |
| 25% decrease in disutilities | 9.95 | $1,952,808 | $196,308 (+2.5%) | 11.14 | $1,412,393 | $126,821 (+2.2%) | |
| Other scenarios of interest | VOC rate: increased by 50% for at-home management | 10.44 | $1,927,427 | $191,519 (−3.6%) | 11.62 | $1,386,810 | $119,326 (−3.8%) |
| Unpaid caregiving: $32,808 per year | 10.20 | $1,952,808 | $191,519 (0.0%) | 11.39 | $1,326,460 | $116,503 (−6.1%) | |
| Caregiver disutility: applied to two caregivers per patient | 10.20 | $1,952,808 | $191,519 (0.0%) | 12.57 | $1,412,393 | $112,320 (−9.5%) | |
| Model settings | Discounting: undiscounted | 21.25 | $1,874,012 | $88,184 (−54.0%) | 22.27 | $966,727 | $43,406 (−65.0%) |
| Discounting: 1.5% per year | 14.34 | $1,882,536 | $131,283 (−31.5%) | 15.50 | $1,193,315 | $76,989 (−37.9%) | |
| Unrelated direct medical costs: $3761 per year | 10.20 | $1,992,453 | $195,407 (+2.0%) | 11.39 | $1,452,038 | $127,533 (+2.8%) | |
| Consumption costs: 80.7% of annual earnings | 10.20 | $1,952,808 | $191,519 (0.0%) | 11.39 | $1,826,232 | $160,399 (+29.3%) | |
diff. difference, Hb hemoglobin, HRQOL health-related quality of life, QALY quality-adjusted life-year, SD standard deviation, sVOE-CR complete resolution of severe vaso-occlusive events, VOC vaso-occlusive crisis, VOE vaso-occlusive event, VOE-CR complete resolution of vaso-occlusive events
aAdditional implementation details and relevant sources for all scenarios are presented in Table S15 in the Supplementary Information
bBaseline age scenarios considered as post hoc subgroups (12–17 years: n = 395 [15.8%]; 18–30 years: n = 1,555 [62.2%]; > 30 years: n = 550 [22.0%])