An Observational Study of Ivacaftor in Patients with Cystic Fibrosis and Selected Non-G551D Gating Mutations in UK, Italy and Netherlands: Healthcare Resource Utilization from the First Interim Analysis of the Vocal Study

Abstract

Objectives: To examine trends in hospital length of stay and total costs in PID patients. MethOds: The latest available 2015 National Inpatient Sample (NIS) data set of hospital admissions from the Healthcare Cost and Utilization Project was utilized in order to determine the number of hospital admissions for patients with PID (identified by ICD-9 for Q1-Q3 and ICD-10 codes for Q4). The LOS and costs were compared by payer type, race and age groups. Results: Based on our inclusion criteria we found 13,393 admissions, representing 0.155% of all hospitalizations. The mean age was 50.04 years (SD: 25.46, Median 57 years). The overall mean LOS was 8.05 days (SD: 13.61, Median 5 days). Majority of the hospitalizations were in patients age 60+ (46%), followed by age 45-59 (20%). Mean (SD) LOS by age: 0-18: 11 days (SD: 25, n= 1725), 18-45: 7.1 days (SD: 10.98, n= 1696), 45-59: 7.11 days (SD: 10.14, n= 2002) and 60+: 7.39 days (SD: 8.48, n= 4622). The mean LOS by payer: Medicare: 7.22 days (SD: 7.82), Medicaid: 9.99 days (SD: 21.48) and Private: 7.94 days (SD: 13.23). The overall mean charges were $90,602

Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review

“…Five (9%) studies were multinational [ 28 , 32 , 33 , 39 , 40 , 89 ]. Thirteen (23%) studies had follow-up times of <12 months [ 38 , 69 , 72 , 78 , 79 , 82 , 84 , 86 , 87 , 88 , 89 , 94 , 102 ], 26 (46%) had follow-up times of 12–23 months [ 28 , 34 , 37 , 39 , 40 , 48 , 49 , 50 , 52 , 53 , 56 , 57 , 59 , 62 , 66 , 67 , 68 , 71 , 73 , 75 , 76 , 81 , 83 , 91 , 95 , 96 , 97 , 98 , 99 ], 14 (25%) had follow-up times of 24–59 months [ 31 , 33 , 51 , 54 , 55 , 61 , 70 , 74 ,…”

“…Thirty-one (54%) studies included both adult and pediatric populations [ 28 , 29 , 30 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 48 , 49 , 50 , 51 , 52 , 53 , 54 , 56 , 62 , 66 , 68 , 69 , 72 , 75 , 76 , 77 , 79 , 80 , 81 , 86 , 87 , 88 , 89 , 90 , 92 , 96 , 98 , 99 , 100 ], whereas 16 (28%) studies included only adult populations [ 32 , 33 , 36 , 37 , 58 , 60 , 61 , 70 , 71 , 74 , 82 , 83 , 84 , 85 , 94 , 95 ,…”

“…There were five (18%) studies that did not report the age of the included population [ 31 , 55 , 73 , 78 , 91 ]. Thirty-nine (68%) studies were restricted to pwCF who had a CFTR gating mutation [ 28 , 31 , 32 , 33 , 36 , 37 , 39 , 40 , 43 , 44 , 45 , 47 , 49 , 51 , 52 , 53 , 54 , 57 , 58 , 59 , 60 , 61 , 62 , 63 , 64 , 65 , 66 , 69 , 70 , 72 , 74 , 76 , 77 , 78 , 79 , 80 , 81 , 83 , 84 , 85 , 86 , 87 , 88 , 89 , 90 , 92 , 93 , 96 , 97 , 98 , 99 , …”

“…Of these, 17 studies had baseline values collected during a period before ivacaftor therapy, whereas 33 studies followed pwCF after starting ivacaftor therapy, with baseline values reported at the time of enrollment or ivacaftor treatment initiation. Of the 50 studies, 11 were large multicenter registry or database studies that included data for ≥50 pwCF [ 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 47 , 52 , 53 ].…”

“…Weight increased from baseline by 0.8–2.8 kg at 1 month [ 42 , 45 , 46 , 96 ] and by 2.8–7.2 kg at 12 months following initiation of ivacaftor [ 37 , 49 , 51 , 96 , 98 , 99 ]. BMI was observed to increase from baseline by 0.2–0.7 kg/m 2 at 1 month [ 42 , 45 , 46 , 95 , 96 ] and by 0.6–2.4 kg/m 2 at 12 months [ 34 , 37 , 39 , 44 , 53 , 66 , 71 , 73 , 75 , 83 , 95 , 96 , 98 , 99 ].…”