1. BURDEN OF ILLNESS IN CYSTIC FIBROSIS: A RETROSPECTIVE ANALYSIS OF MEDICAL EXPENDITURE PANEL SURVEY (MEPS) DATA
Pratyusha Vadagam, MS Candidate,1 Khalid M Kamal, MPharm., PhD,1
Kumar Mukherjee, PhD,2 Jordan R Covvey, PharmD, PhD, BCPS,1 Vincent Giannetti, PhD1
1School of Pharmacy, Duquesne University, Pittsburgh, PA; 2College of Pharmacy, Philadelphia College of Osteopathic Medicine, Suwanee, GA
BACKGROUND
RESULTS
Cystic fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene [1]
CF occurs at a rate of 1 in 3,400 births in the United States (US) affecting 30,000 people nationally and 70,000 globally [1]
Despite the low disease prevalence, the economic burden is substantial with an average disease-related cost of $48,000 per person (2006) [2]
Very few studies have reported burden of illness and health care resource utilization due to CF [2,3]
No recent studies have comprehensively reported the economic burden of CF due to utilization of various medical services like outpatient visits, emergency visits, office visits, home health, prescription drugs, and hospital inpatient stays
The need for current estimates of out-of-pocket (OOP) expenditures and total economic impact of CF warrants further research on the burden of CF care
The overall economic impact of CF on the US population can be calculated using weighted estimates from a nationally representative sample taken from the Medical Expenditure Panel Survey (MEPS) data
Table 1. DEMOGRAPHICS
Table 2. MEDICAL EVENTS
Variables
Cases (N=109)
Age
Mean: 38.2 ± years
Range: 0-82 years
Gender
Male: 39 (35.77%)
Female: 70 (64.22%)
Race
White: 71 (65.14%)
Black: 16 (14.68%)
Hispanic: 17 (15.60%)
Asian: 2 (1.83%)
Others: 3 (2.75%)
Household income
Mean: $59, ± 54,970.57
(95% CI: $49,467.19, $70,340.37)
Marital status
Married: 36 (32.03%)
Not married: 44 (40.36%)
Underage (<16 years): 29 (26.60%)
Insurance coverage
Any private: 73 (66.97%)
Public Only: 35 (32.11%)
Uninsured: 1 (0.92%)
Comorbidities*
High blood pressure
45 (41.28%)
Multiple diagnosis of high blood pressure
39 (35.77%)
Coronary heart disease
10 (9.17%)
Angina
4 (3.66%)
Heart attack (MI)
5 (4.59%)
Other heart disease
20 (18.35%)
Stroke
7 (6.42%)
Emphysema
6 (5.50%)
High cholesterol
37 (33.94%)
Cancer
12 (11%)
Diabetes
11 (10.09%)
Arthritis
33 (30.28%)
Asthma
18 (16.51%)
Medical event
Observations
Total
Total unique observations
2010
2011
2012
2013
2014
Home health 7 2 16 4
Inpatient
stays 1 3 5 11
Office visits 34 37 93 66 53
283 85
Outpatient visits 30 47 9 22
Emergency visits 15 12
Prescription medicines 57 67
182
129
119
554 62
Table 3. EXPENDITURES FROM
Year
Out of pocket expenditure for all medical events ($)
Cumulative expenditure for all medical events ($)
Unweighted
Weighted
2010
1,011.98
15,697,839
16,013.54
195,618,996
2011
3,835.34
40,815,097
46,368.86
413,793,959
2012
12,878.75
255,766,819
156,688.8
2,883,703,057
2013
5,354.42
53,153,619
94,148.59
924,855,899
2014
8,734.59
249,258,850
141,922.4
2,261,164,451
Sum
31,828.58
615,098,138
455,539.7
6,691,134,764
OBJECTIVE
To estimate the burden of illness of cystic fibrosis using MEPS data
METHODS
Data source
The study involved a retrospective analysis of MEPS data from
CF cases were identified using ICD-9-CM diagnosis code for principal diagnosis of CF (277.0)
Criteria for selection of cases
Data analysis
Descriptive analyses were conducted to assess patient demographics, clinical characteristics such as comorbidities and resource utilization related to events in different settings
Unweighted and weighted annual OOP costs and cumulative health care costs were calculated
Statistical analyses were conducted using Statistical Analysis System 9.4 (SAS Institute; Cary, NC)
Not married includes divorced, separated, never married, widowed
* not mutually exclusive
Out of pocket: expenditure paid directly by an individual, Cumulative: expenditure paid by an individual and payments by private insurance, Medicare, Medicaid and other sources
Table 4. OUT OF POCKET EXPENDITURE OF EACH MEDICAL EVENT ( )
Unweighted
Weighted
Mean $ (95% CI) 
Total $
Office visits
87.30 (43.25, )
7,419.71
(39.38, )
105,795,071
Home health
24.50 (-53.46, ) 98
68.75 (-22.50, )
4,377,427
Prescription medicines
(23.58, )
18,328.66
(-23.89, )
433,098,457
Inpatient stays
65.06 (3.72, )
715.73
46.85 (-11.78, )
6,412,171
Outpatient visits
(-65.70, )
4,979.17
(-47.76, )
63,645,296
Emergency visits
23.94 (-14.72, 62.61)
2,87.31
13.14 (-13.96, 40.26)
1,769,716
Sum -
31,828.58
615,098,138
Records in medical conditions file of MEPS data with ICD-9-CM code 277.0
(N=179)
Number of unique records
(N=130)
Number of unique records with no reported usage of any medical service
(N=21)
Mean: mean costs for all the observations of a medical event in a year, Total: sum of costs for all the observations of a medical event in a year, Out of pocket: expenditure paid directly by an individual, CI: confidence interval
Number of unique records with reported usage of at least one medical service
(N=109)
Table 5. CUMULATIVE EXPENDITURE OF EACH MEDICAL EVENT ( )
Unweighted
Weighted
Mean $ (95% CI) 
Total $
Office visits
(342.21, 1,196.85)
65,410.35
(194.80, 1,360.18)
812,971,988
Home health
4, ( , 9,681.97)
17,450
5, (4,221.79, 7,501.73)
373,213,579
Prescription medicines
1, (538.49, 3,227.37)
150,634.2
2, (143.38, 3,948.82)
2,385,010,201
Inpatient stays
13, (3,450.11, 22,611.93)
143,341.3
16,070 ( , 32,402.03)
2,199,126,682
Outpatient visits
3, ( , 7,517.78)
71,917.52
2, ( , 5,893.63)
848,785,772
Emergency visits
(157.57, )
6,786.38
(57.518, 1,012.76)
72,026,542
Sum -
455,539.70
6,691,134,764
Mean: mean costs for all the observations of a medical event in a year, Total: sum of costs for all the observations of a medical event in a year. Cumulative: expenditure paid by an individual and payments by private insurance, Medicare, Medicaid and other sources, CI: confidence interval
DISCUSSION
From , a total of 130 unique CF cases were available, of which 109 (83.8%) reported using at least one CF-related medical service
Over 5 years, unweighted OOP costs was $31,828.58, unweighted cumulative health care costs was $455,539.70, weighted OOP costs was $615,098,138 and weighted total health care costs was $6,691,134,764 [Table 3]
Prescription medications accounted for the highest unweighted and weighted total OOP costs, and also highest unweighted and weighted total cumulative health care costs [Tables 4,5]
With the introduction of novel CFTR therapies into the market, these healthcare expenditure estimates could serve as a guide for managed care organizations in selecting cost effective medications and in setting premiums for insurance plans. The cost estimates from this study could also guide the government in making future decisions for allocation of resources and research funds for CF
Resource utilization can be minimized in the high cost categories by appropriate disease management programs. Providers can focus on identification of misdiagnosis of CF, implementing proper screening techniques in at risk population and patient education
Although implementation of disease intervention programs may add additional costs, future resource utilization can be minimized in CF which could promote cost savings
LIMITATIONS
REFERENCES
MEPS data is self reported and subject to recall bias
As MEPS data is self reported, over or underestimation of health care expenditure is possible
MEPS underreports utilization compared to the National Health Expenditure Accounts (NHEA), which provides the official estimates of health care expenditure in the US
The sample identified from MEPS may not be a true representation of CF population in the US
NIH. What causes cystic fibrosis. Accessed March 5, 2017
Ouyang L, Grosse SD, Amendah DD, Schechter MS. Healthcare expenditures for privately insured people with cystic fibrosis. Pediatric pulmonology Oct 1; 44(10):
Lieu TA, Ray GT, Farmer G, Shay GF. The cost of medical care for patients with cystic fibrosis in a health maintenance organization. Pediatrics Jun 1; 103(6): e72
No financial support was received for the study
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