Model inputs
| Base-case value (range) | Distribution | Reference | |
| Efficacy of ivacaftor | |||
| Patient | |||
| Aged 6–11 years | 10.0 (–) | [5] | |
| Aged ≥12 years | 10.5 (–) | [6] | |
| Relative risk of pulmonary exacerbation | |||
| Aged 6–11 years | 1.0 (–) | [5] | |
| Aged ≥12 years | 0.43 (0.27–0.59) | Log-normal | [6] |
| Relative risk of pulmonary exacerbation hospitalisation | |||
| Aged 6–11 years | 1.0 (–) | [5] | |
| Aged ≥12 years | 0.64 (0.40–1.00) | Log-normal | [6] |
| Average FEV1 % pred for moderate lung disease | |||
| Aged 6–11 years | 30.0 (20.20–39.80) | Gamma | Assumption |
| Aged ≥12 years | 55.0 (45.20–64.80) | Normal | Assumption |
| Transition probability | |||
| Mild to moderate lung disease | Vary based on age (–) | Beta | [16] |
| Mild to severe lung disease | Vary based on age (–) | Beta | [16] |
| Mild to lung transplantation | Vary based on age (–) | Beta | [16] |
| Moderate to severe lung disease | Vary based on age (–) | Beta | [16] |
| Moderate to lung transplantation | Vary based on age (–) | Beta | [16] |
| Severe lung disease to lung transplantation | Vary based on age (–) | Beta | [16] |
| Mortality data | |||
| Mild lung disease to death | Age-specific mortality (–) | [17] | |
| Moderate lung disease to death | 0.030 (0.025–0.375) | Uniform (±25%) | [18] |
| Severe lung disease to death | 0.070 (0.053–0.088) | Uniform (±25%) | [18] |
| Lung transplantation to death | 0.152 (0.139–0.165) | Beta | [19] |
| Healthcare resource utilisation | |||
| Mild lung disease | |||
| Hospitalisation | 0.20 (0–0.894) | Gamma | [21] |
| Outpatient hospitalisation | 0.10 (0–0.447) | Gamma | [21] |
| Clinic visits | 7.00 (0–16.690) | Gamma | [21] |
| Probability of patients using pancreatic enzymes | 0.89 (–) | [21] | |
| Probability of patients using intravenous antibiotics | 0.02 (–) | [21] | |
| Probability of patients using DNase | 0.36 (–) | [21] | |
| Moderate lung disease | |||
| Hospitalisation | 0.40 (0–1.488) | Gamma | [21] |
| Outpatient hospitalisation | 0.30 (0–1.541) | Gamma | [21] |
| Clinic visits | 7.00 (0–15.790) | Gamma | [21] |
| Probability of patients using pancreatic enzymes | 0.84 (–) | [21] | |
| Probability of patients using intravenous antibiotics | 0.49 (–) | [21] | |
| Probability of patients using DNase | 0.72 (–) | [21] | |
| Severe lung disease | |||
| Hospitalisation | 1.70 (0–4.399) | Gamma | [21] |
| Outpatient hospitalisation | 1.40 (0–5.058) | Gamma | [21] |
| Clinic visits | 10.00 (0–22.100) | Gamma | [21] |
| Probability of patients using pancreatic enzymes | 0.95 (–) | [21] | |
| Probability of patients using intravenous antibiotics | 0.71 (–) | [21] | |
| Probability of patients using DNase | 0.71 (–) | [21] | |
| Probability of double lung transplantation | 0.964 (0.960–0.968) | Beta | [24] |
| Cost US$ | |||
| Mild lung disease | |||
| Hospitalisation | 2406.61 (0–18 522.86) | Gamma | [21] |
| Clinic visit | 2406.61 (0–6730.48) | Gamma | [21] |
| DNase | 3008.26 (0–13 228.32) | Gamma | [21] |
| Outpatient antibiotics | 802.20 (0–3946.84) | Gamma | [21] |
| Pancreatic enzymes | 3008.26 (0–8904.45) | Gamma | [21] |
| Other medications | 1002.75 (0–3361.23) | Gamma | [21] |
| Moderate lung disease | |||
| Hospitalisation | 7821.47 (0–60 101.04) | Gamma | [21] |
| Clinic visit | 2206.06 (0–8310.82) | Gamma | [21] |
| DNase | 5214.32 (0–20 568.48) | Gamma | [21] |
| Outpatient antibiotics | 1604.41 (0–13 613.38) | Gamma | [21] |
| Pancreatic enzymes | 3008.26 (0–7909.72) | Gamma | [21] |
| Other medications | 1203.30 (0–2374.52) | Gamma | [21] |
| Severe lung disease | |||
| Hospitalisation | 56 154.20 (0–224 392.18) | Gamma | [21] |
| Clinic visit | 6217.07 (0–17 616.37) | Gamma | [21] |
| DNase | 10 027.54 (0–31 646.90) | Gamma | [21] |
| Outpatient antibiotics | 9425.88 (0–53 843.85) | Gamma | [21] |
| Pancreatic enzymes | 2406.61 (0–7516.64) | Gamma | [21] |
| Other medications | 2807.71 (0–13 420.85) | Gamma | [21] |
| Single lung | |||
| 30 days pre-transplant | 10 889.39 (8167.05–13 611.74) | Uniform (±25%) | [22] |
| Procurement | 77 283.01 (57 962.26–96 603.76) | Uniform (±25%) | [22] |
| Hospital transplant admission | 320 232.87 (240 174.66–400 291.09) | Uniform (±25%) | [22] |
| Physician fee during transplantation | 35 416.97 (26 562.73–44 271.22) | Uniform (±25%) | [22] |
| 180 days post-transplantation | 124 435.16 (93 326.37–155 543.95) | Uniform (±25%) | [22] |
| Outpatient immunosuppressant and other medication | 25 056.19 (18 792.14–31 320.23) | Uniform (±25%) | [22] |
| Follow-up in months 7–12 after transplantation | 65 459.75 (49 094.81–81 824.69) | Uniform (±25%) | [23] |
| Follow-up for the following year | 108 899.04 (81 674.28–136 123.80) | Uniform (±25%) | [23] |
| Double lung | |||
| 30 days pre-transplant | 22 624.57 (16 968.43–28 280.72) | Uniform (±25%) | [22] |
| Procurement | 95 467.25 (71 600.43–119 334.06) | Uniform (±25%) | [22] |
| Hospital transplant admission | 484 736.79 (363 522.59–605 920.98) | Uniform (±25%) | [22] |
| Physician fee during transplantation | 59 521.67 (44 641.25–74 402.08) | Uniform (±25%) | [22] |
| 180 days post-transplantation | 150 760.01 (113 070.01–188 450.02) | Uniform (±25%) | [22] |
| Out-patient immunosuppressant and other medication | 29 813.69 (22 360.27–37 267.11) | Uniform (±25%) | [22] |
| Follow-up in months 7–12 after transplantation | 65 459.75 (49 094.81–81 824.69) | Uniform (±25%) | [23] |
| Follow-up for the following year | 108 899.04 (81 674.28–136 123.80) | Uniform (±25%) | [23] |
| Ivacaftor per tablet | 426.72 (320.04–533.40) | Uniform (±25%) | [14] |
| Utility | |||
| Patients with mild lung disease | 0.803 (0.752–0.854) | Beta | [15] |
| Patients with moderate lung disease | 0.749 (0.708–0.790) | Beta | [15] |
| Patients with severe lung disease | 0.688 (0.639–0.737) | Beta | [15] |
| Patients with lung transplantation | 0.810 (0.732–0.888) | Beta | [15] |
FEV1: forced expiratory volume in 1 s.