Table 2.

Base-case and scenario analysis results for iptacopan monotherapy vs anticomplement C5 therapy

StrategyCost (million $)Incremental
cost (million $)		Effectiveness (QALYs)Incremental
effectiveness (QALYs)		% cost-effective at WTP threshold of $50 000 per QALY% cost-effective at WTP threshold of $100 000 per QALY% cost-effective at WTP threshold of $150 000 per QALY
US societal perspective        
Base case∗         
Iptacopan 9.52  12.6  100 100 100 
Anti-C5 13.5 4.01 10.8 −1.78 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$4 100  000 (3 250 000-4 990 000), $4 190 000 (3 330 000-5 100 000), $4 280 000 (3 390 000-5 210 000). 
Scenario (5-y time horizon)        
Iptacopan 2.34  3.08  100 100 100 
Anti-C5 3.33 0.99 2.65 −0.43 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$1 010 000 (803 000-1 230 000), $1 030 000 (822 000-1 260 000), $1 050 000 (837 000-1 290 000) 
Scenario (PRINCE utilities)        
Iptacopan 9.52  13.6  100 100 100 
Anti-C5 13.5 4.01 12.5 −1.11 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$4 070 000 (3 240 000-4 960 000), $4 120 000 (3 290 000-5 030 000), $4 180 000 (3 340 000-5 120 000) 
Scenario (GBD 2019 utilities)        
Iptacopan 9.52  15.7  100 100 100 
Anti-C5 13.5 4.01 14.3 −1.38 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$4 080 000 (3 250 000-4 960 000), $4 150 000 (3 320 000-5 040 000), $4 220 000 (3 390 000-5 110 000) 
Scenario (average wholesale price)        
Iptacopan 12.8  12.6  100 100 100 
Anti-C5 15.0 2.21 10.8 −1.78 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$2 300 000 (1 450 000-3 180 000), $2 390 000 (1 530 000-3 290 000), $2 480 000 (1 590 000-3 410 000) 
Scenario (iptacopan discontinuation)        
Iptacopan 11.1  11.8  100 100 100 
Anti-C5 13.5 2.48 10.9 −1.02 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$2 530 000 (1 890 000-3 190 000), $2 580 000 (1 920 000-3 260 000), $2 630 000 (1 960 000-3 330 000) 
Scenario (Iptacopan discontinuation, 5-y time horizon)        
Iptacopan 2.43  3.04  100 100 100 
Anti-C5 3.33 0.90 2.65 −0.39 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$920 000 (726 000-1 130 000), $940 000 (741 000-1 150 000), $959 000 (756 000-1 180 000) 
Scenario (individual-level, trial-specific patient data transition probability matrix)        
Iptacopan 9.28  12.6  100 100 100 
Anti-C5 11.7 2.38 10.9 −1.70 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$2 470 000 (2 150 000-2 810 000), $2 550 000 (2 230 000-2 910 000), $2 640 000 (2 320 000-3 000 000) 
Scenario (individual-level, trial-specific patient data transition probability matrix and health state utilities)        
Iptacopan 9.28  13.7  100 100 100 
Anti-C5 11.7 2.38 10.9 −2.83 0 0 0 
iNMB at WTP thresholds $50 000, $100 000, $150 000 per QALY (95% CI)
$2 520 000 (2 200 000-2 860 000), $2 660 000 (2 330 000-3 010 000), $2 800 000 (2 480 000-3 170 000) 
US health care system perspective        
Base case        
Iptacopan 9.52  12.6  100 100 100 
Anti-C5 13.4 3.84 10.8 −1.78 0 0 0 
iNMB at WTP thresholds $50 000, $100 000, $150 000 per QALY (95% CI)
$3 930 000 (3 090 000-4 810 000), $4 020 000 (3 170 000-4 920 000), $4 110 000 (3 230 000-5 040 000) 
Scenario (5-y time horizon)        
Iptacopan 2.34  3.08  100 100 100 
Anti-C5 3.29 0.95 2.65 −0.43 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$969 000 (760 000-1 190 000), $991 000 (781 000-1 210 000), $1 010 000 (797 000-1 240 000) 
StrategyCost (million $)Incremental
cost (million $)		Effectiveness (QALYs)Incremental
effectiveness (QALYs)		% cost-effective at WTP threshold of $50 000 per QALY% cost-effective at WTP threshold of $100 000 per QALY% cost-effective at WTP threshold of $150 000 per QALY
US societal perspective        
Base case∗         
Iptacopan 9.52  12.6  100 100 100 
Anti-C5 13.5 4.01 10.8 −1.78 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$4 100  000 (3 250 000-4 990 000), $4 190 000 (3 330 000-5 100 000), $4 280 000 (3 390 000-5 210 000). 
Scenario (5-y time horizon)        
Iptacopan 2.34  3.08  100 100 100 
Anti-C5 3.33 0.99 2.65 −0.43 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$1 010 000 (803 000-1 230 000), $1 030 000 (822 000-1 260 000), $1 050 000 (837 000-1 290 000) 
Scenario (PRINCE utilities)        
Iptacopan 9.52  13.6  100 100 100 
Anti-C5 13.5 4.01 12.5 −1.11 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$4 070 000 (3 240 000-4 960 000), $4 120 000 (3 290 000-5 030 000), $4 180 000 (3 340 000-5 120 000) 
Scenario (GBD 2019 utilities)        
Iptacopan 9.52  15.7  100 100 100 
Anti-C5 13.5 4.01 14.3 −1.38 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$4 080 000 (3 250 000-4 960 000), $4 150 000 (3 320 000-5 040 000), $4 220 000 (3 390 000-5 110 000) 
Scenario (average wholesale price)        
Iptacopan 12.8  12.6  100 100 100 
Anti-C5 15.0 2.21 10.8 −1.78 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$2 300 000 (1 450 000-3 180 000), $2 390 000 (1 530 000-3 290 000), $2 480 000 (1 590 000-3 410 000) 
Scenario (iptacopan discontinuation)        
Iptacopan 11.1  11.8  100 100 100 
Anti-C5 13.5 2.48 10.9 −1.02 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$2 530 000 (1 890 000-3 190 000), $2 580 000 (1 920 000-3 260 000), $2 630 000 (1 960 000-3 330 000) 
Scenario (Iptacopan discontinuation, 5-y time horizon)        
Iptacopan 2.43  3.04  100 100 100 
Anti-C5 3.33 0.90 2.65 −0.39 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$920 000 (726 000-1 130 000), $940 000 (741 000-1 150 000), $959 000 (756 000-1 180 000) 
Scenario (individual-level, trial-specific patient data transition probability matrix)        
Iptacopan 9.28  12.6  100 100 100 
Anti-C5 11.7 2.38 10.9 −1.70 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$2 470 000 (2 150 000-2 810 000), $2 550 000 (2 230 000-2 910 000), $2 640 000 (2 320 000-3 000 000) 
Scenario (individual-level, trial-specific patient data transition probability matrix and health state utilities)        
Iptacopan 9.28  13.7  100 100 100 
Anti-C5 11.7 2.38 10.9 −2.83 0 0 0 
iNMB at WTP thresholds $50 000, $100 000, $150 000 per QALY (95% CI)
$2 520 000 (2 200 000-2 860 000), $2 660 000 (2 330 000-3 010 000), $2 800 000 (2 480 000-3 170 000) 
US health care system perspective        
Base case        
Iptacopan 9.52  12.6  100 100 100 
Anti-C5 13.4 3.84 10.8 −1.78 0 0 0 
iNMB at WTP thresholds $50 000, $100 000, $150 000 per QALY (95% CI)
$3 930 000 (3 090 000-4 810 000), $4 020 000 (3 170 000-4 920 000), $4 110 000 (3 230 000-5 040 000) 
Scenario (5-y time horizon)        
Iptacopan 2.34  3.08  100 100 100 
Anti-C5 3.29 0.95 2.65 −0.43 0 0 0 
iNMB at WTP thresholds of $50 000, $100 000, $150 000 per QALY (95% CI)
$969 000 (760 000-1 190 000), $991 000 (781 000-1 210 000), $1 010 000 (797 000-1 240 000) 

Iptacopan monotherapy is a cost-saving treatment option across all scenarios.

CI, credible interval; iNMB, incremental net monetary benefit; QALY, quality-adjusted life year; WTP, willingness-to-pay.

∗

Patient time lost from C5 inhibitor use in base case: 730 hours from ravulizumab and 2920 hours from eculizumab.

View Large
This Feature Is Available To Subscribers Only

or Create an Account

Close Modal
Close Modal