Base case average microsimulation results for 18-y-old until death time horizon, gene therapy infusion age 18 y old, in US dollars and QALYs
| Outcome . | On-demand . | Prophylaxis . | Gene therapy . | |||
|---|---|---|---|---|---|---|
| Standard half-life FIX . | Extended half-life FIX . | Standard half-life FIX . | Extended half-life FIX . | Standard half-life FIX . | Extended half-life FIX . | |
| Approach A . | Approach B . | Approach C . | Approach D . | Approach E . | Approach F . | |
| Costs | $11 596 617 | $7 917 721 | $15 109 058 | $20 324 299 | $6 293 502 | $7 315 914 |
| (11 586 467- 11 606 767)* | (7 911 165- 7 924 277)* | (15 101 499-1 5 116 617)* | (20 314 604- 20 333 993)* | (6 288 817- 6 298 187)* | (7 309 727- 7 322 100)* | |
| QALYs | 11.81 | 12.20 | 20.95 | 21.57 | 23.00 | 23.04 |
| (11.80-11.81)* | (12.20-12.21)* | (20.94-20.95)* | (21.56-21.58)* | (22.99-23.01)* | (23.03-23.05)* | |
| Cost/QALY | $981 932 | $648 994 | $721 196 | $942 248 | $273 631 | $317 531 |
| Gene ICER† | Dominant‡ | Dominant‡ | Dominant‡ | Dominant‡ | Reference§ | $25 560,300‖ |
| Outcome . | On-demand . | Prophylaxis . | Gene therapy . | |||
|---|---|---|---|---|---|---|
| Standard half-life FIX . | Extended half-life FIX . | Standard half-life FIX . | Extended half-life FIX . | Standard half-life FIX . | Extended half-life FIX . | |
| Approach A . | Approach B . | Approach C . | Approach D . | Approach E . | Approach F . | |
| Costs | $11 596 617 | $7 917 721 | $15 109 058 | $20 324 299 | $6 293 502 | $7 315 914 |
| (11 586 467- 11 606 767)* | (7 911 165- 7 924 277)* | (15 101 499-1 5 116 617)* | (20 314 604- 20 333 993)* | (6 288 817- 6 298 187)* | (7 309 727- 7 322 100)* | |
| QALYs | 11.81 | 12.20 | 20.95 | 21.57 | 23.00 | 23.04 |
| (11.80-11.81)* | (12.20-12.21)* | (20.94-20.95)* | (21.56-21.58)* | (22.99-23.01)* | (23.03-23.05)* | |
| Cost/QALY | $981 932 | $648 994 | $721 196 | $942 248 | $273 631 | $317 531 |
| Gene ICER† | Dominant‡ | Dominant‡ | Dominant‡ | Dominant‡ | Reference§ | $25 560,300‖ |
Results are presented in boldface font for 3 treatment strategies: on-demand treatment, prophylaxis, and gene therapy, 2 approaches each: standard half-life FIX and extended half-life FIX. Approaches are labeled A to F. Supplemental Table 1 provides further definitions of standard health economics terms, and the supplemental Methods provides details regarding the methodologic approach.
95% Confidence intervals are presented between parentheses in regulat face font for all costs and QALYs.
ICER of gene therapy reference treatment approach vs alternative in costs per added QALY.
Gene therapy reference treatment approach is dominant (less costly and yields more QALYs).
Gene therapy approach with the lower cost per QALY was used as the reference and compared with the 5 other approaches.
Gene therapy reference treatment approach yields less QALYs but remains cost-effective considering a $150 000/QALY threshold.