Healthcare expenditures for privately insured US patients with cystic fibrosis, 2010-2016

Pediatr Pulmonol. 2018 Dec;53(12):1611-1618. doi: 10.1002/ppul.24178. Epub 2018 Oct 31.


Objectives: Published cost estimates for cystic fibrosis (CF) are based on older data and do not reflect increased use of specialty drugs in recent years. We assessed recent trends in healthcare expenditures for CF patients in the United States (US) with employer-sponsored health insurance.

Methods: The study is a retrospective analysis of claims data for privately insured individuals aged 0-64 years who were continuously enrolled in non-capitated plans for at least 1 calendar year during 2010-2016. Mean annual expenditures during a calendar year were calculated for individuals who met a claims-based CF case definition. Average annual growth rates were calculated through linear regression of the natural logarithm of annual expenditures.

Results: The annual CF prevalence was 1.1-1.4 per 10 000 adults and 2.9-3.0 per 10 000 children. Average spending adjusted for inflation nearly doubled from roughly $67 000 per patient in 2010 and 2011 to approximately $131 000 per patient in 2016. Inflation-adjusted spending on outpatient and inpatient care increased by 0.5% and 2.5% per year, respectively, whereas pharmaceutical spending increased by 20.2% per year. Virtually all of the growth in pharmaceutical spending was accounted for by spending on specialty drugs; inflation-adjusted spending on other medications increased by 1.3% per year. The annual growth rate in pharmaceutical spending rose by 33.1% during 2014-2016, the years during which lumacaftor/ivacaftor was introduced.

Conclusions: Per-patient expenditures for privately-insured patients with CF almost doubled during 2010-2016; specialty drugs were largely responsible for this increase, with a major contribution from new, genotype-targeted CFTR modulator medications.

Keywords: cystic fibrosis; drugs; health expenditures; precision medicine; rare diseases.

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Cost of Illness
  • Cystic Fibrosis / drug therapy
  • Cystic Fibrosis / economics*
  • Fees, Pharmaceutical
  • Female
  • Health Benefit Plans, Employee*
  • Health Expenditures*
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Middle Aged
  • Retrospective Studies
  • United States
  • Young Adult