Evaluating the financial impact of utilizing recombinant porcine factor VIII or recombinant FVIIa for patients with acquired hemophilia A

Figures & data

Table 1. Patient level baseline demographics and other patient characteristics [7].

Patient Characteristics	rpFVIII (n = 25)		rFVIIa (n = 114)		Total Population (n = 166)
	Mean	SD	Mean	SD	Mean	SD
Charlson Comorbidity Index score	2.32	2.29	1.74	1.97	1.83	2.02
Age	74.96	6.58	74.43	10.60	74.5	10.08
	Observed Difference* between rpFVIII (n = 25) and rFVIIa (n = 141)				N	%
Male	+0.88%				105	63.25%
Race
White	−13.3%				125	75.3%
Black	+13.1%				28	16.9%
Other	+2.58%				13	7.8%
Region
Midwest	+10.0%				39	23.5%
Northeast	+2.0%				37	22.3%
South	+10.0%				59	35.5%
West	−22%				31	18.7%
CCI Component Prevalence at Baseline
Cerebrovascular Disease	−1.93%				16	9.64%
Congestive Heart Failure	−1.73%				29	17.47%
Chronic Pulmonary Disease	−5.99%				35	21.08%
Dementia	−1.67%				-	-
Diabetes	+7.69%				29	17.47%
Hemiplegia or Paraplegia	+1.87%				-	-
HIV/AIDS	+0.71%				-	-
Myocardial Infarction	+6.78%				17	10.24%
Metastatic Solid Tumor	+5.87%				-	-
Peptic Ulcer Disease	−1.67%				-	-
Peripheral Vascular Disease	−10.89%				22	13.25%
Renal Disease	−4.82%				40	24.10%
Rheumatologic Disease	+7.23%				23	13.86%
Mild Liver Disease	+11.74%				-	-
Moderate/Severe Liver Disease	−1.42%				-	-
Other Comorbidities
Autoimmune disorders^†	+12.77%				148	89.16%
Medications^‡
Treated with factor VIII	−11.73%				29	17.47%
Treated with aPCC	−12.82%				38	22.89%

*Calculated as the percent of the rpFVIII cohort with the characteristic of interest minus the percent of the rFVIIa cohort with the characteristic of interest. Positive values indicate higher prevalence in the rpFVIII cohort.

^†Autoimmune lymphoproliferative syndrome, hemophagocytic lymphohistiocytosis, Langerhans cell histiocytosis, acute lymphoblastic leukemia, chronic lymphocytic leukemia, multiple myeloma, Wiskott–Aldrich syndrome, non-Hodgkin’s lymphoma, rheumatoid arthritis, Sicca (Sjögren) syndrome, connective tissue diseases, systemic lupus erythematosus, thyroiditis, antiphospholipid syndrome, diseases of the blood and blood-forming organs, and certain other disorders involving the immune mechanism.

^‡Patients were assessed for the presence of any claims indicating treatment with medications of interest and/or presence of relevant comorbidities at any time from the pre-index period to the end of their follow-up. “-” Values with <11 patients masked to comply with data privacy best practices. Charlson Comorbidity Index = CCI; aPCC = Activated prothrombin complex concentrate; rFVIIa = activated recombinant Factor VII; rpFVIII = recombinant porcine FVIII.

Table 2. Admission level findings [7].

Outcome	rpFVIII		rFVIIa
Total Number of Admissions	40		180
Mean Length of Stay (LOS) (SD)	17.2 (14.4)		15.2 (16.6)
	rpFVIII vs. rFVIIa Admissions
	Unadjusted Results		Adjusted* Results
	Probability	p-value	Probability	p-value
Death during most recent admission	−12.5%	0.075	−13.0%	0.059
Death within 90 days post-discharge of most recent admission	−20.2%	0.025	−21.4%	0.014

Note: rFVIIa admissions served as the reference group. For example, a − 12.5 probability of death during the most recent index admission means that patients treated with rpFVIII had a 12.5% lower probability of dying during the hospital admission than patients treated with rFVIIa.

*Calculated using multivariate regression, adjusted results were controlled for age, gender, and Charloson Comorbidity Index score.

rFVIIa = activated recombinant Factor VII; rpFVIII = recombinant porcine FVIII.

Table 3. Cost outcomes for admissions with treatment.

Cost Outcomes	rpFVIII	rFVIIa	Overall Hospitalizations
	(n = 40)	(n = 180)	(n = 220)
	Mean
	(SD)
Reimbursed amount to hospital	$503,641	$371,026	$394,751
	($539,779)	($97,490)	($588,601)
Charged amount from hospital	$ 1,819,019	$1,536,877	$1,588,176
	($ 1,689,816)	($2,677,688)	($2,526,030)
Average Medicare Cost-to-Charge ratio (CCR)	0.27 (0.06)	0.26 (0.09)	0.26 (0.08)
Cost to hospital (Charged amount * CCR)	$ 452,092	$ 335,082	$ 356,357
	($369,503)	($ 469,103)	($ 454,119)
Impact/Margin (Reimbursed amount minus Cost to Hospital)	+$51,549	+$ 35,944	+$ 38,394

(+) indicate postive margins , i.e. hospital cost was less than the amount reimbursed to the hospital. Margins were calculated as reimbursed amount to hospital minus cost to hospital. (SD)=standard deviation

Table 4. Sensitivity analysis for admissions with treatment.

Sensitivity	rpFVIII		rFVIIa
CCR	Hospital Cost*	Margin**	Hospital Cost	Margin**
0.2	$ 363,804	$139,837	$ 307,375	$ 63,650
0.25	$ 454,755	$48,886	$ 384,219	($13,193)
0.3	$ 545,706	($42,065)	$ 461,063	($90,037)
0.35	$ 636,657	($133,016)	$ 537,907	($166,881)
0.4	$ 727,607	($223,967)	$ 614,751	($243,725)
0.45	$ 818,558	($314,918)	$ 691,595	($320,569)
0.47	$ 854,939	($351,298)	$ 722,332	($351,306)
0.475	$ 864,034	($360,393)	$ 730,017	($358,991)
0.5	$ 909,509	($405,869)	$ 768,439	($397,413)

*Hospital cost = sensitivity CCR x average charged [rpFVIII: $1,819,019; rFVIIa: $1,536,877] (see Table 2).

**Margin = Average reimbursed amount [rpFVIII:503,641; rFVIIa:$371,026] (see Table 2) – Hospital Cost*. Values in parentheses indicate negative margins.

Batt K, Bullano M, Schultz BG, et al. Evaluating the impact of recombinant porcine factor VIII compared with activated recombinant factor VII on acquired hemophilia A outcomes. Future Rare Dis. 2022;2(2):FRD23.

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