TABLE 1

Model inputs

Base-case value (range)DistributionReference
Efficacy of ivacaftor
 Patient
  Aged 6–11 years10.0 (–)[5]
  Aged ≥12 years10.5 (–)[6]
 Relative risk of pulmonary exacerbation
  Aged 6–11 years1.0 (–)[5]
  Aged ≥12 years0.43 (0.27–0.59)Log-normal[6]
 Relative risk of pulmonary exacerbation hospitalisation
  Aged 6–11 years1.0 (–)[5]
  Aged ≥12 years0.64 (0.40–1.00)Log-normal[6]
 Average FEV1 % pred for moderate lung disease
  Aged 6–11 years30.0 (20.20–39.80)GammaAssumption
  Aged ≥12 years55.0 (45.20–64.80)NormalAssumption
Transition probability
 Mild to moderate lung diseaseVary based on age (–)Beta[16]
 Mild to severe lung diseaseVary based on age (–)Beta[16]
 Mild to lung transplantationVary based on age (–)Beta[16]
 Moderate to severe lung diseaseVary based on age (–)Beta[16]
 Moderate to lung transplantationVary based on age (–)Beta[16]
 Severe lung disease to lung transplantationVary based on age (–)Beta[16]
Mortality data
 Mild lung disease to deathAge-specific mortality (–)[17]
 Moderate lung disease to death0.030 (0.025–0.375)Uniform (±25%)[18]
 Severe lung disease to death0.070 (0.053–0.088)Uniform (±25%)[18]
 Lung transplantation to death0.152 (0.139–0.165)Beta[19]
Healthcare resource utilisation
 Mild lung disease
  Hospitalisation0.20 (0–0.894)Gamma[21]
  Outpatient hospitalisation0.10 (0–0.447)Gamma[21]
  Clinic visits7.00 (0–16.690)Gamma[21]
  Probability of patients using pancreatic enzymes0.89 (–)[21]
  Probability of patients using intravenous antibiotics0.02 (–)[21]
  Probability of patients using DNase0.36 (–)[21]
 Moderate lung disease
  Hospitalisation0.40 (0–1.488)Gamma[21]
  Outpatient hospitalisation0.30 (0–1.541)Gamma[21]
  Clinic visits7.00 (0–15.790)Gamma[21]
  Probability of patients using pancreatic enzymes0.84 (–)[21]
  Probability of patients using intravenous antibiotics0.49 (–)[21]
  Probability of patients using DNase0.72 (–)[21]
 Severe lung disease
  Hospitalisation1.70 (0–4.399)Gamma[21]
  Outpatient hospitalisation1.40 (0–5.058)Gamma[21]
  Clinic visits10.00 (0–22.100)Gamma[21]
  Probability of patients using pancreatic enzymes0.95 (–)[21]
  Probability of patients using intravenous antibiotics0.71 (–)[21]
  Probability of patients using DNase0.71 (–)[21]
  Probability of double lung transplantation0.964 (0.960–0.968)Beta[24]
Cost US$
 Mild lung disease
  Hospitalisation2406.61 (0–18 522.86)Gamma[21]
  Clinic visit2406.61 (0–6730.48)Gamma[21]
  DNase3008.26 (0–13 228.32)Gamma[21]
  Outpatient antibiotics802.20 (0–3946.84)Gamma[21]
  Pancreatic enzymes3008.26 (0–8904.45)Gamma[21]
  Other medications1002.75 (0–3361.23)Gamma[21]
 Moderate lung disease
  Hospitalisation7821.47 (0–60 101.04)Gamma[21]
  Clinic visit2206.06 (0–8310.82)Gamma[21]
  DNase5214.32 (0–20 568.48)Gamma[21]
  Outpatient antibiotics1604.41 (0–13 613.38)Gamma[21]
  Pancreatic enzymes3008.26 (0–7909.72)Gamma[21]
  Other medications1203.30 (0–2374.52)Gamma[21]
 Severe lung disease
  Hospitalisation56 154.20 (0–224 392.18)Gamma[21]
  Clinic visit6217.07 (0–17 616.37)Gamma[21]
  DNase10 027.54 (0–31 646.90)Gamma[21]
  Outpatient antibiotics9425.88 (0–53 843.85)Gamma[21]
  Pancreatic enzymes2406.61 (0–7516.64)Gamma[21]
  Other medications2807.71 (0–13 420.85)Gamma[21]
 Single lung
  30 days pre-transplant10 889.39 (8167.05–13 611.74)Uniform (±25%)[22]
  Procurement77 283.01 (57 962.26–96 603.76)Uniform (±25%)[22]
  Hospital transplant admission320 232.87 (240 174.66–400 291.09)Uniform (±25%)[22]
  Physician fee during transplantation35 416.97 (26 562.73–44 271.22)Uniform (±25%)[22]
  180 days post-transplantation124 435.16 (93 326.37–155 543.95)Uniform (±25%)[22]
  Outpatient immunosuppressant and other medication25 056.19 (18 792.14–31 320.23)Uniform (±25%)[22]
  Follow-up in months 7–12 after transplantation65 459.75 (49 094.81–81 824.69)Uniform (±25%)[23]
  Follow-up for the following year108 899.04 (81 674.28–136 123.80)Uniform (±25%)[23]
 Double lung
  30 days pre-transplant22 624.57 (16 968.43–28 280.72)Uniform (±25%)[22]
  Procurement95 467.25 (71 600.43–119 334.06)Uniform (±25%)[22]
  Hospital transplant admission484 736.79 (363 522.59–605 920.98)Uniform (±25%)[22]
  Physician fee during transplantation59 521.67 (44 641.25–74 402.08)Uniform (±25%)[22]
  180 days post-transplantation150 760.01 (113 070.01–188 450.02)Uniform (±25%)[22]
  Out-patient immunosuppressant and other medication29 813.69 (22 360.27–37 267.11)Uniform (±25%)[22]
  Follow-up in months 7–12 after transplantation65 459.75 (49 094.81–81 824.69)Uniform (±25%)[23]
  Follow-up for the following year108 899.04 (81 674.28–136 123.80)Uniform (±25%)[23]
 Ivacaftor per tablet426.72 (320.04–533.40)Uniform (±25%)[14]
Utility
 Patients with mild lung disease0.803 (0.752–0.854)Beta[15]
 Patients with moderate lung disease0.749 (0.708–0.790)Beta[15]
 Patients with severe lung disease0.688 (0.639–0.737)Beta[15]
 Patients with lung transplantation0.810 (0.732–0.888)Beta[15]
  • FEV1: forced expiratory volume in 1 s.