Table 1.

Summary of profiled CE models of CAR-T therapies

Author/year	Extrapolation technique	Model approach	OS parametric distribution	Model time horizon	Median trial follow-up durations	Intervention	Comparator	Incremental life years
Adult DLBCL
Marchetti 2018 [53]	Advanced	Mixture cure	NR	Lifetime	27.1 months§	Axi-cel	Salvage chemotherapy	NR
NICE 2018 (Axi) [42]	Advanced	Mixture cure	Weibull	Lifetime	27.1 months§	Axi-cel	Salvage chemotherapy	Redacted
NICE 2018 (Tis) [43]	Advanced	Mixture cure	Log-normal	Lifetime	Redacted‖	Tis-cel	Salvage chemotherapy	Redacted
					28.6 months¶
Roth 2018 [38]	Advanced	Mixture cure	Gompertz	Lifetime	27.1 months§	Axi-cel	Salvage chemotherapy	6.89
CADTH 2019 (Axi) [45]	Advanced	Mixture cure	Redacted	44 years	27.1 months§	Axi-cel	Salvage chemotherapy	NR
CADTH 2019 (Tis) [46]	Advanced	Weighted average	Weighted average**	20 years	Redacted ‖	Tis-cel	Salvage chemotherapy	3.29
					28.6 months¶
MSAC 2019 (Tis) [49]	Advanced	Spline	NA	Lifetime	NR‖	Tis-cel	Salvage chemotherapy	1.47
Karampampa 2020 [54]	Advanced	Mixture cure	NR	Lifetime	39.1 months	Axi-cel	Salvage chemotherapy	4.92–5.16
Liu 2020 [40]	Advanced	Mixture cure	NR	Lifetime	27.1 months§	Axi-cel	Tis-cel	4.26
MSAC 2020 (Axi) [48]	Advanced	Mixture cure	Weibull	Lifetime	27.1 months§	Axi-cel	Salvage chemotherapy	4.53
Sierra 2020 [51]	Advanced	Mixture cure	NR	Lifetime	27.1 months§	Axi-cel	Salvage chemotherapy	5.76
ICER 2018 (DLBCL) [35]	Standard or other	Standard PSM	Log-normal	Lifetime	27.1 months§	Axi-cel	Salvage chemotherapy	4.12
Lin 2019 (adult) [34]	Standard or other	Optimization	NA	Lifetime	27.1 months§	Axi-cel	Salvage chemotherapy	8.15
					NR	Tis-cel	Salvage chemotherapy	4.60
Pediatric ALL
NICE 2018 (ALL) [41]	Advanced	Mixture cure	Exponential	Lifetime	13.1 months*	Tis-cel	Salvage chemotherapy	Redacted
					NR†		Blinatumomab	Redacted
					NR‡
Ribera Santasusana 2020 [50]	Advanced	Weighted average	Weighted average**	Lifetime	13.1 months*	Tis-cel	Salvage chemotherapy	10.10
					NR†
					NR‡
ICER 2018 (ALL) [35]	Standard or other	Standard PSM	Log-normal	Lifetime	13.1 months*	Tis-cel	Clofarabine	7.91
Lin 2019 (pediatric) [34]	Standard or other	Optimization	NA	Lifetime	13.1 months*	Tis-cel	Blinatumomab	12.05
							Clofarabine combination	12.05
							Clofarabine monotherapy	13.00
MSAC 2019 (ALL) [47]	Standard or other	Standard PSM	Log-normal	Lifetime	13.1 months*	Tis-cel	Blinatumomab	5.20
					NR†		Salvage chemotherapy	6.83
Furzer 2020 [44]	Standard or other	Microsimulation	NR	60 years	13.1 months*	Tis-cel	Salvage chemotherapy	2.14
Sarkar 2020 [39]	Standard or other	Microsimulation	NA	Lifetime	13.1 months*	Tis-cel	Salvage chemotherapy	NR
					NR†
Thielen 2020 [52]	Standard or other	Standard PSM	Log-normal	Lifetime	13.1 months*	Tis-cel	Blinatumomab	10.84
					NR†		Clofarabine combination	11.55
					NR‡		Clofarabine monotherapy	13.27

ALL acute lymphoblastic leukemia, Axi axicabtagene ciloleucel, CADTH Canadian Agency for Drugs and Technologies in Health, CAR-T chimeric antigen receptor therapy, CE cost-effectiveness, DLBCL diffuse large B cell lymphoma, ICER Institute for Clinical and Economic Review, MSAC Medical Services Advisory Committee, NA not applicable, NICE National Institute for Health and Care Excellence, NR not reported, PSM partitioned-survival model, QALYs quality adjusted life years, Tis tisagenlecleucel

*ELIANA

†ENSIGN

‡B2101J

§ZUMA−1

‖JULIET

¶UPENN

**Extrapolated curve was estimated on the basis of the weighted average of parametric functions (exponential, Weibull, Gompertz, log-normal, log-logistic, gamma) and spline curves (with one, two, three, or four nodes)