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. 2021 Jan 28;5(2):e524. doi: 10.1097/HS9.0000000000000524

Table 2.

Overview of Cost-effectiveness Analysis Publications.

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.