Table 1. Summary of Variables Applied in the Cost-effectiveness Model.
| Variable | Value (SD) [Range] | Distribution Assumed in PSA | Source |
|---|---|---|---|
| Total No. | 21 | NA | Russell et al,9 2017a |
| HS1: moderate VI | 19 | ||
| HS2: severe VI | 24 | ||
| HS3: profound VI | 29 | ||
| HS4: CF | 29 | ||
| HS5: HM/LP/NLP, % | 0 | ||
| Age, y | 15 (10.9) [4-44] | Uniform | Russell et al,9 2017 |
| Progression rate during SC (natural history) | 1 [2-1000] | Uniform | Chung et al, al,18 2019 |
| VA | −0.16 (0.13) [−0.41 to 0.08] | Normal | Russell et al,9 2017 |
| VF | 378.7 (119.0) [145.5-612.0] | Normal | Russell et al,9 2017 |
| Reduction in long-term treatment effect after year 3 (%) | Lifetime (0) [0-100] | NA | Assumption |
| VI mortality rate | Christ et al,22 2014 | ||
| HS1: moderate VI hazard rate | 1.08 [1.02-1.15] | ||
| HS2: severe VI hazard rate | 1.18 [1.05-1.32] | ||
| HS3: profound VI hazard rate | 1.18 [1.05-1.32] | ||
| HS4: CF hazard rate | 1.18 [1.05-1.32] | ||
| HS5: HM/LP/NLP hazard rate | 1.18 [1.05 to 1.32] | ||
| HS1: moderate VI | 0.71 (0.04) [0.63-0.78] | β | Lloyd et al,23 2019 |
| HS2: severe VI | 0.62 (0.02) [0.58-0.65] | β | |
| HS3: profound VI | 0.52 (0.03) [0.46-0.57] | β | |
| HS4: CF | 0.35 (0.03) [0.30-0.40] | β | |
| HS5: HM/LP/NLP | 0.15 (0.05) [0.06-0.24] | β | |
| HS1: moderate VI | 8818 (882) [7055-10 582] | γ | Brown et al,27 2016 |
| HS2: severe VI | 11 573 (1157) [9259-13 888] | γ | |
| HS3: profound VI | 12 655 (1266) [10 124-15 186] | γ | |
| HS4: CF | 13 737 (1374) [10 990-16 485] | γ | |
| HS5: HM/LP/NLP | 13 737 (1374) [10 990-16 485] | γ | |
| Age <18 y | Jensen et al,30 2018 | ||
| HS1: moderate VI | 10 005 (1000) [8004-12 006] | γ | |
| HS2: severe VI | 56 883 (5688) [45 507-68 260] | γ | |
| HS3: profound VI | 56 883 (5688) [45 507-68 260] | γ | |
| HS4: CF | 74 877 (7488) [59 901-89 852] | γ | |
| HS5: HM/LP/NLP | 74 877 (7488) [59 901-89 852] | γ | |
| Age ≥18 y | Jensen et al,30 2018 | ||
| HS1: moderate VI | 24 191 (2419) [19 353-29 029] | γ | |
| HS2: severe VI | 60 226 (6023) [48 181-72 271] | γ | |
| HS3: profound VI | 60 226 (6023) [48 181-72 271] | γ | |
| HS4: CF | 95 184 (9518) [76 147 -114 221] | γ | |
| HS5: HM/LP/NLP | 95 184 (9518) [76 147-114 221] | γ | |
| Discount rate, annual, costs, % | 3.0 | NA | Sanders et al,31 2016 |
| Discount rate, annual, utilities, % | 3.0 | NA | |
| Voretigene neparvovec-rzyl, price per injection, $b | 854 535 | NA |
Abbreviations: CF, count fingers; EQ-5D, EuroQol 5-dimension, 5-level standard for valuing quality of life; HM, hand motion; HS, health state; LP, light perception; NA, not applicable; NLP, no light perception; PSA, probabilistic sensitivity analyses; SC, standard care; VA, visual acuity; VF, visual field; VI, visual impairment.
a
Analysis of baseline visual acuity and visual field data from AAV2-hRPE65v2-301.19
b
Cost of voretigene neparvovec-rzyl based on published wholesale acquisition cost, cost of administration based on Medicare 2018 average national reimbursement for the Comprehensive Ambulatory Payment Classification for a level 2 intraocular procedure and Current Procedural Terminology 67036, vitrectomy, mechanical, pars plana approach.