Burden of illness: direct and indirect costs among persons with hemophilia A in the United States

Figures & data

Table 1. Direct medical cost components.

Type	Cost data source(s)
Clotting factor and bypassing agent	Factor unit cost is estimated using average sales price from the payment allowance limit for Medicare Part B26
Healthcare service	Cost for CPT code from the Medicare
Comprehensive care	fee schedule relative value units25
Clinician visit	(RVUs)
Physical therapy
Social worker
Psychology
Hospitalization	Based on the average cost for hospitalization with certain discharge diagnosis determined from Healthcare Cost and Utilization Project22. Adjusted by cost-to-charge ratio23
Emergency department	Medical Expenditure Panel Survey (MEPS) reports30
Outpatient procedure	Cost for CPT code from the Medicare fee schedule RVUs25
Lab test	Cost for CPT code from the Medicare fee schedule RVUs26
Other haemophilia-related medication(s)	Wholesale acquisition costs27

Table 2. Baseline characteristics.

Baseline characteristics^a	Included (n = 222)	Excluded (n = 107)	p Value
Age, mean (SD)	21.1 (14.9)	22.5 (15.7)	0.4788
Older than 18 years, n (%)	112 (50.5)	52 (48.6)	0.7529
Hemophilia severity, n (%)
Mild	45 (20.3)	37 (34.6)	0.0049
Moderate	31 (14.0)	5 (4.7)	0.0114
Severe	146 (65.8)	65 (60.7)	0.3740
Employment status, n (%)^b,c
Full-Time	97 (43.7)	53 (49.5)	0.3192
Part-Time	54 (24.3)	20 (18.7)	0.2517
Not Employed	71 (32.0)	34 (31.8)	0.9700
Married/with a partner, (%)^b	125 (56.3)	68 (63.6)	0.2112
Education: >12 years, N (%)^b	160 (72.1)	66 (61.7)	0.0569
Household income: >$20,000, n (%)^b,c	173 (77.9)	83 (77.6)	0.9417
Race/Ethnicity, n (%)^c
White/Non-Hispanic	161 (72.5)	52 (48.6)	<0.0001
Black/Non-Hispanic	11 (5.0)	5 (4.7)	0.9113
Hispanic	26 (11.7)	33 (30.8)	<0.0001
Other^d	24 (10.8)	17 (15.9)	0.1915
Health insurance, n (%)^c
No insurance	6 (2.7)	6 (5.6)	0.1880
Partial-year insurance coverage	13 (5.9)	6 (5.6)	0.9279
Full-year insurance coverage	196 (88.3)	94 (87.9)	0.9084
Comorbidities, n (%)
HIV/AIDS	31 (27.7)	13 (25.5)	0.7704
HCV	78 (72.9)	35 (70.0)	0.7065
Use prophylaxis, n (%)	97 (43.7)	47 (43.9)	0.9684
Current inhibitors, n (%)	8 (3.6)	5 (4.7)	0.6409

Abbreviations: SD: standard deviation; HIV/AIDS: human-immunodeficiency-virus and acquired immune deficiency syndrome; HCV: hepatitis C virus

^aChi-square tests were used for comparisons of categorical variables. Wilcoxon signed-rank tests were used for comparisons of continuous variables.

^bFor patient or parent if patient age <18 years.

^cData do not add up to n = 329 because of missing data.

^dOther races include American Indian, Alaska Native, Asian/Pacific Islander and others.

Table 3. Average annual medically related absenteeism and number of bleeding episodes by age and hemophilia severity (n = 222).

	Children			p Value	Adults			p Value
	Mild (n = 22)	Moderate (n = 17)	Severe (n = 71)		Mild (n = 23)	Moderate (n = 14)	Severe (n = 75)
Annual missed work/school days
All causes	5.3 (8.9)	6.5 (11.3)	8.0 (16.3)	0.3394	6.2 (10.1)	5.6 (12.6)	21.0 (50.2)	0.4109
Due to hemophilia	3.3 (7.5)	4.7 (8.7)	5.6 (12.2)	0.1785	4.7 (9.2)	4.3 (11.6)	19.4 (49.9)	0.7208
Parents’ missed work days  due to child’s hemophilia	3.6 (8.8)	3.2 (8.9)	3.1 (10.0)	0.9719	–	–	–	–
Annual number of bleeding episodes	4.1 (6.9)	9.1 (8.2)	6.4 (7.9)	0.0085	4.5 (10.0)	8.6 (11.8)	20.2 (14.5)	<0.0001

Events were annualized and reported as mean (standard deviation). Wilcoxon signed-rank tests were used for the comparisons.

Figure 1. Average healthcare resource utilization and bleeding episodes among patients with severe haemophilia. Persons with inhibitors (n = 10) were excluded. Length of hospital stay only applies to patients who were hospitalized (n = 26). *Statistically significant differences at p < 0.05 were observed between patients who used a clotting factor on demand vs prophylactically.

Table 4. Average hemophilia-related costs per person per year by hemophilia severity and treatment patterns.

12-month costs^a	Total (n = 212)	Mild (n = 44)	Moderate (n = 31)	Severe (n = 137)
				On-demand (n = 51)	Prophylaxis (n = 86)
Total direct costs^b,c	185,257 [113,857] (190,621)	53,907 [5863] (140,080)	75,320 [32,687] (121,600)	184,518 [125,385] (160,758)	292,525 [272,892] (186,739)
Clotting factor costs^b,c	178,400 [107,319] (188,588)	47,649 [5501] (137,489)	70,438 [32,391] (116,855)	170,037 [118,259] (151,846)	289,172 [272,236] (186,502)
Average percent attributable  to total costs^b,c	0.80	0.54	0.74	0.84	0.94
Other medical costs^c	6857 [878] (22,451)	6258 [605] (11,875)	4882 [1000] (9154)	14,481 [1502] (41,881)	3353 [500] (8352)
Total indirect costs	10,076 [233] (20,773)	5195 [0] (16,877)	9043 [0] (21,439)	16,952 [301] (26,068)	8867 [376] (17,959)
Total costs (direct + indirect)^b,c	195,332 [139,571] (194,179)	59,101 [7519] (149,369)	84,363 [61,837] (121,433)	201,471 [143,431] (164,377)	301,392 [286,198] (188,977)

^aCosts were reported as mean [median] (standard deviation) during the 12-month period. All costs were inflated to 2011 US dollars. Direct costs were inflated using the CPI for Medical Care. Indirect costs were inflated using average hourly compensation data from the Bureau of Labor Statistics. Costs for persons with inhibitors (n = 10) were not reported in the table.

^bStatistically significant differences at p < 0.05 were observed among the mild, moderate and severe groups.

^cStatistically significant differences at p < 0.05 were observed between patients who used clotting factor on demand vs prophylactically.

Figure 2. Average hemophilia-related costs per person per year by age group. Costs were reported as mean in 2011 US dollars. Costs for persons with inhibitors (n = 10) were excluded. *Statistically significant differences at p < 0.05 were observed between children and adults.

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