Table 2.
References | Indication, Treatment | Base-case Settings | Scenario Analysis | Total Costs | Total Effects in QALYs | ICER: CAR T-cell vs |
---|---|---|---|---|---|---|
Lin et al34 | pALL, tisagenlecleucel | Perspective: healthcare | Yes, 5-y relapse-free survival rates (ie, 40%-0%) | (2017 USD) | Clo-M: 3.12 | (USD/QALY) |
Horizon: lifetime | Clo-M: 314 000 | Clo-C: 3.52 | Best-case scenario (40% 5-y relapse-free survival rate) | |||
Discount rate (costs/effects): 3%/3% | Clo-C: 374 000 | Blina: 3.57 | Clo-M: 61 315, Clo-C: 43 103, Blina: 50 712 | |||
Blina: 282 000 | CAR T-cell: 8.74 | |||||
CAR T-cell: 599 000 | ||||||
Whittington et al35 | pALL, tisagenlecleucel | Perspective: healthcare | Yes, other discount rates, different survival curve fitting, future healthcare cost (included/not included | (2017 USD) | Clo-M: 2.10 | (USD/QALY) |
Horizon: lifetime | Clo-M: 337 256 | CAR T-cell: 9.28 | Base-case scenario: 46 000 | |||
Discount rate (costs/effects): 3%/3% | CAR T-cell: 666 754 | |||||
Sarkar et al36 | pALL, tisagenlecleucel | Perspective: healthcare | No | (2017 USD) | Clo-C: 8.58 | (USD/QALY) |
Horizon: lifetime | Clo-C: 440 600 | CAR T-cell: 16.76 | Payer perspective: 64 600 | |||
Discount rate (costs/effects): 3%/3% | CAR T-cell: 968 800 | |||||
Walton et al37 | pALL, tisagenlecleucel | Perspective: NA | No | (2017 GBP) | Salvage chemo: NA | (GBP/QALY) |
Horizon: lifetime | Salvage chemo: NA | Blin: NA | Deterministic: Salvage chemo 45 397, Blina: 27 732 | |||
Discount rate (costs/effects): 3.5%/3.5% | Blina: NA | CAR T-cell: NA | ||||
CAR T-cell: NA | ||||||
Furzer et al38 | pALL, tisagenlecleucel | Perspective: Public insurer | Yes, long-term cure rates varying between 10% and 40% | (2018 USD) | Comparator: 5.05 | (USD/QALY) |
Horizon: 60 y | Comparator (combination of chemo and HSCT): 86 597 | Optimistic scenario: 53 933 | ||||
Discount rate (costs/effects): 1.5%/1.5% | CAR T-cell: 442 098 | CAR T-cell: 14.90 | ||||
Thielen et al39 | pALL, tisagenlecleucel | Perspective: Societal | Yes, different perspectives, shorter plateau phase, different time horizons, alternative standardized mortality rate input, vial sharing assumed, longer duration of IVIG administration, different parametric extrapolation models resulting in different cure rates | (2018 EUR) | Clo-M: 0.74 | (EUR/QALY) |
Horizon: lifetime | Clo-M: 160 803 | Clo-C: 1.70 | Base-case | |||
Discount rate (costs/effects): 4%/1.5% | Clo-C: 193 920 | Blina: 2.25 | Clo-M: 36 378 EUR/QALY | |||
BlinaL 267 259 | CAR T-cell: 11.26 | Clo-C: 31 052 EUR/QALY | ||||
CAR T-cell: 552 679 | Blina: 31 682 EUR/QALY | |||||
Roth et al40 | DLBCL, axicabtagene ciloleucel | Perspective: healthcare | Yes, patients in long-term remission experience 10% or 20% lower survival compared with age-matched US general population | (2018 USD) | R-DHAP: 1.13 | (USD/QALY) |
Horizon: lifetime | R-DHAP: 172 737 | CAR T-cell: 7.67 | Base-case scenario: 58 146 | |||
Discount rate (costs/effects): 3%/3% | CAR T-cell: 552 921 | |||||
Lin et al45 | DLBCL, tisagenlecleucel, and axicabtagene ciloleucel | Perspective: healthcare | Yes, PFS at 5 y: Axicabtagene: 40% to 20% and tisagenlecleucel: 35% to 15% | (2018 USD) | Combination: R-DHAP, R-GDP, R-GEMOX, R-ICE, SCT: 1.78 | (USD/QALY) |
Horizon: lifetime | Combination: R-DHAP, R-GDP, R-GEMOX, R-ICE, SCT: 169 000 | Axicabtagene ciloleucel: 5.50 | Axicabtagene ciloleucel vs combination (40% 5-y progression-free survival): 129 570 | |||
Discount rate (costs/effects): 3%/3% | Axicabtagene ciloleucel: 651 000 | Tisagenlecleucel: 3.92 | Tisagenlecleucel vs combination (35% 5-y progression-free survival): 168 224 | |||
Tisagenlecleucel: 529 000 | ||||||
Whittington et al41 | DLBCL, axicabtagene ciloleucel | Perspective: public payer care | Yes, different extrapolation of OS and PFS curves, different perspectives, different time horizons | (Year not clear USD) | R-DHAP: 3.37 | (USD/QALY) |
Horizon: lifetime | R-DHAP: 151 200 | CAR T-cell: 9.19 | Public payer perspective, standard parametric: 230 900 | |||
Discount rate (costs/effects): 3%/3% | CAR T-cell: 554 700 |
CAR T = chimeric antigen receptor T cell; DLBCL = diffuse large B-cell lymphoma; EUR = Euro; GBP = Pound sterling; HSCT = autologous haematopoietic stem cell transplantation; ICER = incremental cost-effectiveness ratio; IVIG = intravenous immunoglobulin; pALL = pediatric acute lymphoblastic leukemia; NA = not available; OS = overall survival; PFS = progression-free survival; QALY = quality-adjusted life year; R-DHAP = rituximab dexamethasone cytarabine cisplatin; R-GDP = rituximab gemcitabine dexamethasone cisplatin; R-GEMOX = rituximab gemcitabine oxaliplatin; R-ICE = rituximab ifosfamide carboplatin etoposide; SCT = stem cell transplantation; USD = United States dollar.