Cost-effectiveness analysis of lumacaftor and ivacaftor combination for the treatment of patients with cystic fibrosis in the United States

Sharma, Dolly; Xing, Shan; Hung, Yu-Ting; Caskey, Rachel N.; Dowell, Maria L.; Touchette, Daniel R.

doi:10.1186/s13023-018-0914-3

Table 2 Summary of key model inputs: probabilities, costs and utilities. Transition probabilities are presented for each model transition, including those from a Markov state (e.g. Mild CF) to transition states (e.g. Pulmonary Exacerbation)

From: Cost-effectiveness analysis of lumacaftor and ivacaftor combination for the treatment of patients with cystic fibrosis in the United States

Variable	Base-case	One-way		Source
Variable	Base-case	Low	High	Source
Probabilities
Mild to Death	Age-specific mortality for CF population			[3]
Mild to Pulmonary Exacerbation	0.3213	0.2571	0.3856	[16]
Pulmonary Exacerbation in Mild to Death	0.001	0.0008	0.0012	Assumption
Pulmonary Exacerbation in Mild to Moderate	0.0697	0.0558	0.0837	[14, 15]
No Pulmonary Exacerbation in Mild to Moderate	0.0307	0.0245	0.0368	[14, 15]
Moderate to Death	Age-specific mortality for CF population			[3]
Moderate to Pulmonary Exacerbation	0.5748	0.4599	0.6898	[16]
Pulmonary Exacerbation in moderate to Death	0.001	0.0008	0.0012	Assumption
Pulmonary Exacerbation in Moderate to Severe	0.0617	0.0493	0.074	[14, 15]
No Pulmonary Exacerbation in Moderate to Severe	0.0279	0.0223	0.0335	[14, 15]
Severe to Death	Age-specific mortality for CF population			[3]
Severe to Pulmonary Exacerbation	0.6794	0.5435	0.8153	[16]
Pulmonary Exacerbation in severe to death	0.2174	0.1739	0.2609	[18]
Transplant to Death	0.297	0.2376	0.3564	[17]
Pulmonary Exacerbation in Severe to Transplant	0.078	0.0624	0.0937	[17]
No Pulmonary Exacerbation in Severe to Transplant	0.078	0.0624	0.0937	[17]
Post-transplant to Death	Calculated year-wise			[19]
Lumacaftor/Ivacaftor:
Moderate to Mild without Pulmonary Exacerbation	0.0935	0.0748	0.1123	[5, 14, 15]
Severe to Moderate without Pulmonary Exacerbation	0.5517	0.4414	0.6621	[5, 14, 15]
Moderate to Mild with Pulmonary Exacerbation	0.1045	0.0836	0.1254	[5, 14, 15]
Severe to Moderate with Pulmonary Exacerbation	0.5455	0.4364	0.6545	[5, 14, 15]
Relative risk for Pulmonary Exacerbation (Lumacaftor/Ivacaftor vs Placebo)	0.74	0.592	0.89	[5]
Costs (USD-2016)
Lumacaftor/ivacaftor	188,660.43	150,928.34	226,392.51	[9, 20]
Mean cost of Pulmonary Exacerbation in Mild	2575.97	2575.97	30,949.79	[13]
Mean cost of Pulmonary Exacerbation in Moderate	8371.90	8371.90	41,969.47	[13]
Mean cost of Pulmonary Exacerbation in Severe	52,646.40	52,646.40	123,385.36	[13]
Mild	7566.91	5121.17	30,267.65	[13]
Moderate	9981.89	6709.60	39,927.54	[13]
Severe	17,226.80	8688.16	68,907.21	[13]
Transplant	1,056,002.23	844,801.78	1,267,202.67	[21]
Post-transplant	87,945.85 (1st year)	70,356.68	105,535.02	[21, 22]
Post-transplant	86,332.56 (2nd year onwards)	69,066.05	103,599.07	[22]
Utilities
Mild	0.86	0.77	0.94	[23]
Moderate	0.81	0.72	0.89	[23]
Severe	0.64	0.57	0.7	[23]
Post-transplant	0.83	0.74	0.91	[23]
Pulmonary Exacerbation	− 0.17	− 0.15	−0.19	[23]

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