Healthcare resource utilization associated with ivacaftor use in patients with cystic fibrosis

J Med Econ. 2016 Sep;19(9):845-51. doi: 10.1080/13696998.2016.1178125. Epub 2016 Apr 27.


Objective: Ivacaftor was approved in 2012 to treat patients with cystic fibrosis (CF) with specific CFTR gene mutations. The objective of this analysis was to analyze the impact of ivacaftor on health resource utilization through analysis of claims data.

Methods: Patients diagnosed with CF aged ≥6 years prescribed ivacaftor between January 1, 2012 and July 31, 2014 with ≥12 months of continuous insurance coverage prior to and following the prescription were identified. All-cause and CF-specific healthcare resource utilization during the pre- and post-prescription periods and ivacaftor adherence levels were studied.

Results: The 79 identified patients had a mean age of 20.8 years, and 54% were female. The proportion of patients with inpatient admissions (all-cause and CF-related) was significantly higher in the pre index compared to post index period (p ≤ 0.05). Mean ivacaftor medication possession ratio was 0.8 (SD = 0.3), and 73% of patients had a medication possession ratio >0.80.

Limitations: Only a small number of patients met the inclusion criteria. Additionally, claims data may contain errors or inconsistencies and cannot be used to determine if medications were taken as prescribed.

Conclusions: Ivacaftor therapy was associated with significant reductions in hospitalizations along with high rates of adherence to treatment over 12 months.

Keywords: Adherence; Healthcare resource utilization; Ivacaftor: Cystic fibrosis; Medication possession ratio; Pulmonary exacerbation.

MeSH terms

  • Adolescent
  • Adult
  • Age Factors
  • Aminophenols / economics*
  • Aminophenols / therapeutic use
  • Child
  • Chloride Channel Agonists / economics*
  • Chloride Channel Agonists / therapeutic use
  • Costs and Cost Analysis
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / economics*
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Female
  • Health Expenditures / statistics & numerical data
  • Health Services / economics*
  • Health Services / statistics & numerical data*
  • Humans
  • Insurance, Health / statistics & numerical data
  • Male
  • Middle Aged
  • Mutation
  • Quinolones / economics*
  • Quinolones / therapeutic use
  • Retrospective Studies
  • Sex Factors
  • Young Adult


  • Aminophenols
  • CFTR protein, human
  • Chloride Channel Agonists
  • Quinolones
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • ivacaftor