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

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.

Precision Medicine In Action: The Impact Of Ivacaftor On Cystic Fibrosis-Related Hospitalizations.

Cystic fibrosis is a life-threatening genetic disease that causes severe damage to the lungs. Ivacaftor, the first drug that targeted the underlying defect of the disease caused by specific mutations, is a sterling example of the potential of precision medicine. Clinical trial and registry studies showed that ivacaftor improved outcomes and reduced hospitalizations. Our study used US administrative claims data to assess the real-world effectiveness of ivacaftor. Comparing twelve-month rates before and after starting the use of ivacaftor among people who initiated therapy during 2012-2015, we found that overall and cystic fibrosis-related inpatient admissions fell by 55 percent and 81 percent, respectively. There was a comparable reduction in inpatient spending. Ivacaftor appears to be effective for multiple mutations that cause the disease, as suggested by the fact that during the study period, ivacaftor's use was extended to nine additional mutations in 2014. Examination of evidence from clinical trial, clinical care, and administrative data sources is important for understanding the real-world effectiveness of precision medicines such as ivacaftor.

U.S. trends in computed tomography use and diagnoses in emergency department visits by patients with symptoms suggestive of pulmonary embolism, 2001-2009.

OBJECTIVES: Using computed tomography (CT) to evaluate patients with chest symptoms is common in emergency departments (EDs). This article describes recent trends of CT use in U.S. EDs for patients presenting with symptoms common to acute pulmonary embolism (PE). METHODS: The 2001-2009 National Hospital Ambulatory Medical Care Survey (NHAMCS), a nationally representative survey of U.S. ED encounters, was used for data collection. Patients with at least one of three complaints (chest pain, dyspnea, or hemoptysis) were categorized into the chest symptom study (CSS) group. The yearly increases in CT use for the complaints were tabulated first, then linear regression analysis was used to calculate average rates of increases in CT use between 2001 and 2007, the years where CT use increased, for the overall population and among specific subgroups. The ratios of the number of visits when CT was ordered and there was a target diagnosis relative to the total number of visits with CT in the CSS group (diagnosis/CT ratio) were calculated for PE and pneumonia. RESULTS: Annual CT rates for the CSS group increased from 2.6% in 2001 to 13.2% in 2007, subsequently leveling off at approximately 12.5% in 2008 and 2009. The average growth rate of CT use for the CSS group was 28.1% (95% confidence interval [CI] = 20.9% to 35.7%) per year between 2001 and 2007. Testing rates for all subgroups increased. The lowest growth rate was among Hispanic patients, whose CT rates grew 14.2% (95% CI = 5.7% to 23.5%) per year. The highest growth rate was in nonurban hospitals, at 43.1% (95% CI = 15.2% to 77.8%) per year. Patients triaged as nonurgent received the fewest CTs, compared to those who should be seen in 2 hours or less. With regard to sources of payment, the self-pay subgroup experienced the highest rate of increase at 35.1% (95% CI = 18.6% to 53.9%). The PE diagnosis/CT ratio from 2002 to 2009 was 2.7% for the CSS group. The pneumonia diagnosis/CT ratio grew from 5.8% in 2002 to 2005 to 7.8% in 2006 to 2009. CONCLUSIONS: Computed tomography use in ED visits by patients with chest symptoms increased dramatically from 2001 to 2007 and seems to have leveled off in subsequent years. The low PE diagnosis-to-CT ratio suggests that EDs may need to promote evidence-based use of CT.

National trends in emergency department use, care patterns, and quality of care of older adults in the United States.

OBJECTIVES: To describe trends in use of emergency departments (EDs) of older adults, reasons for visits, resource use, and quality of care. DESIGN: Analysis of the National Hospital Ambulatory Medical Care Survey. SETTING: U.S. emergency departments from 2001 to 2009. PARTICIPANTS: Individuals aged 65 and older visiting U.S. EDs. MEASUREMENTS: Emergency departments (ED) visits by patients aged 65 and older were identified, and demographic, clinical, and resource use characteristics and outcomes were assessed. RESULTS: From 2001 to 2009, annual visits increased from 15.9 to 19.8 million, a 24.5% increase. Numbers of outpatients grew less than hospital admissions (20.2% vs 33.1%); intensive care unit admissions increased 131.3%. Reasons for visits were unchanged during the study; the top complaints were chest pain, dyspnea, and abdominal pain. Resource intensity grew dramatically: computed tomography 167.0%, urinalyses 87.1%, cardiac monitoring 79.3%, intravenous fluid administration 59.8%, blood tests 44.1%, electrocardiogram use 43.4%, procedures 38.3%, and radiographic imaging 36.4%. From 2005 to 2009, magnetic resonance imaging use grew 84.6%. The proportion receiving a potentially inappropriate medication decreased from 9.6% in 2001 to 4.9% in 2009, whereas the proportion seen in the ED, discharged, and subsequently readmitted to the hospital rose from 2.0% to 4.2%. CONCLUSION: Older adults accounted for 156 million ED visits in the United States from 2001 to 2009, with steady increases in visits and resource use across the study period. Hospital admissions grew faster than outpatient visits. If changes in primary care do not affect these trends, facilities will need to plan to accommodate increasingly greater demands for ED and hospital services.