Table 1. . Model inputs: base-case, deterministic sensitivity analysis and probabilistic sensitivity analysis.

Parameter	Base-case value (mean/ percentage)	Deterministic sensitivity analysis		Probabilistic sensitivity analysis	Source	Ref.
		Low value	High value	Distribution and values
Discount rates, %
Discount rate for costs (per year)	3.0	0.0	6.0	Not included	ICER guidelines	[62]
Discount rate for health outcomes (per year)	3.0	0.0	6.0		ICER guidelines	[62]
Baseline patient characteristics
Mean age (years)	44.5	35.6	53.4	Normal (μ = 44.5, σ = 4.54)	PRINCE trial baseline characteristics	[47,63]
Proportion of females, %	45.28	32.23	58.67	Beta (α = 24, β = 29)	PRINCE trial baseline characteristics	[63]
Mean weight (kg)	83.86	67.09	100.63	Normal (μ = 83.86, σ = 8.56)	CDC Vital and Health Statistics	[64]
Health state utilities
Hb normalized	0.869	0.782	0.956	Beta (μ = 0.869, σ = 0.039)	Calculations based on PRINCE trial patients' level data
Hb not normalized	0.820	0.738	0.902	Beta (μ = 0.820, σ = 0.037)
Transfusion required	0.818	0.736	0.900	Beta (μ = 0.818, σ = 0.038)
General population utilities
Age 18–29 years	0.922	Not included		Not included	Sullivan, 2006	[65]
Age 30–39 years	0.901
Age 40–49 years	0.871
Age 50–59 years	0.842
Age 60–69 years	0.823
Age 70–79 years	0.790
Age 80+ years	0.736
Treatment costs
Price per package, $
Pegcetacoplan	4403.84	Not included		Not included	REDBOOK	[66]
Eculizumab	6523.00
Ravulizumab	6404.00
Dosage distributions, %
Pegcetacoplan – 1080 mg twice weekly	100	Not included		Dirichlet (x1 = 100.00%, α1 = 35, x2 = 0%, α2 = 0)	Prescribing information	[32,42]
Pegcetacoplan – 1080 mg every 3 days	0
Eculizumab – 900 mg every 2 weeks	100			Dirichlet (x1 = 100.00%, α1 = 121, x2 = 0.00%, α2 = 0, x3 = 0.00%, α3 = 0, x4 = 0.00%, α4 = 0)
Eculizumab – 900 mg every 11 days	0
Eculizumab – 1200 mg every 2 weeks	0
Eculizumab – 1500 mg every 2 weeks	0
Ravulizumab – 40–60 kg	30.23	21.61	45.34	Dirichlet (x1 = 30.23%, α1 = 3165, x2 = 33.47%, α2 = 3504, x3 = 36.31%, α3 = 3802)	Distribution based on mean weight	[24]
Ravulizumab – 60–100 kg	33.47	23.93	26.22
Ravulizumab – 100+ kg	36.31	28.44	54.46
Total cost per cycle, $
Supportive treatment	4137.55	3310.04	4965.06	Gamma (μ = 4137.55, σ = 445.86)	REDBOOK PRINCE trial	[47,63,66]
Administration costs
Pegcetacoplan	134.96	107.97	161.96	Gamma (μ = 134.96, σ = 14.54)	CMS Fee Schedule	[67]
IV administration cost (first hour)	140.16	112.13	168.19	Gamma (μ = 140.16, σ = 15.10)
IV administration cost (subsequent hours)	29.76	23.81	35.71	Gamma (μ = 29.76, σ = 3.21)
Administration times, minutes
Eculizumab – maintenance dose	35	Not included		Not included	Prescribing information	[32]
Eculizumab – additional observation time after administration	60
Ravulizumab – loading dose, 40–60 kg	114				Prescribing information	[34]
Ravulizumab – loading dose, 60–100 kg	102
Ravulizumab – loading dose, 100+ kg	108
Ravulizumab – maintenance dose, 40–60 kg	140
Ravulizumab – maintenance dose, 60–100 kg	120
Ravulizumab – maintenance dose, 100+ kg	132
Ravulizumab – additional observation time after administration	60
Vaccine costs, $
Meningitis (A, C, Y, W–135)	70.97	56.78	85.17	Gamma (μ = 70.97, σ = 7.65)	CDC Vaccine Price List	[68]
Meningitis (B)	103.43	82.75	124.12	Gamma (μ = 103.43, σ = 11.15)
PCV13	137.22	109.78	164.66	Gamma (μ = 137.22, σ = 14.79)
PPSV23	149.90	119.92	179.88	Gamma (μ = 149.90, σ = 16.15)
Monitoring
Number of hematologist visits per cycle – hemoglobin normalized	1	Not included		Not included	Clinical experts
Number of hematologist visits per cycle – hemoglobin not normalized	1
Number of hematologist visits per cycle – transfusion required	13
Hematologist visit cost, $	134.96	107.97	161.96	Gamma (μ = 134.96, σ = 14.54)	CMS Fee Schedule	[67]
Blood tests related parameters
Number of blood tests per cycle – hemoglobin normalized	2	Not included		Not included	Clinical experts
Number of blood tests per cycle – hemoglobin not normalized	2
Number of blood tests per cycle – transfusion required	4
Blood test cost, $	7.77	6.22	9.32	Gamma (μ = 7.77, σ = 0.84)	CMS Fee Schedule	[67]
Blood transfusions
Total number – initial	2.65	2.12	3.18	Normal (μ = 2.65, σ = 0.27)	Calculations based on PRINCE trial data	[63]
Increment per cycle	0.2	0.16	0.24	Normal (μ = 0.20, σ = 0.02)	Clinical experts
Maximum number in 1 cycle	8.17	6.54	9.80	Normal (μ = 8.17, σ = 0.83)	Calculations based on PRINCE trial data	[63]
QALY loss per transfusion	-0.002	-0.002621108	-0.001747405	Beta (μ,σ) (μ = -0.002, σ = 0.0004)	Calculations based on PRINCE trial patients' level data	[63]
Cost per transfusion, $	2744.32	2195.46	3293.19	Gamma (μ = 2744.32, σ = 295.73)	Cheng, 2021	[2]
AEs
AEs probability – pegcetacoplan – breakthrough hemolysis	4.3%	3.44%	5.15%	Beta (μ = 0.0429, σ = 0.0041)	PRINCE trial data
AEs probability – eculizumab – breakthrough hemolysis	10.7%	8.56%	10.27%	Beta (μ = 0.1070, σ = 0.0109)	Matching adjusted indirect comparison	[69]
AEs probability – eculizumab – major adverse Vascular Events	0.8%	0.66%	0.80%	Beta (μ = 0.0083, σ = 0.0014)
AEs probability – ravulizumab – breakthrough hemolysis	4.0%	3.20%	3.84%	Beta (μ = 0.0400, σ = 0.0038)
AEs probability – ravulizumab – major adverse Vascular Events	1.6%	1.28%	1.54%	Beta (μ = 0.0160, σ = 0.0020)
AEs QALY lost – breakthrough hemolysis	0.0006	0.0005	0.0006	Beta (μ = 0.0006, σ = 0.0001)	O'Connell, 2020	[58]
AEs QALY lost – major adverse vascular events	0.0006	0.0005	0.0006	Beta (μ = 0.0006, σ = 0.0001)	Sullivan, 2006	[65]
AEs treatment cost – major adverse vascular events	$24,190	$19,352.18	$23,222.62	Gamma (μ = 24,190.23, σ = 2,606.72)	Dasta, 2015	[70]

AE: Adverse event; IV: Intravenous; QALY: Quality-adjusted life-year.