Systematic literature review of economics analysis on treatment of mild-to-moderate bleeds with aPCC versus rFVIIa

Figures & data

Figure 1.  Flow diagram of the results of the search strategy.

Table 1.  Comparative economic studies of aPCC and rFVIIa in the treatment of a mild-to-moderate bleeding episode in patients with hemophilia and inhibitors.

	Odeyemi et al.19	Odeyemi et al.20	Chung et al.21	Putnam et al.22	Dundar et al.23	Joshi et al.24	Ozelo et al.25	Carlsson et al.26	You et al.27
Study type	CMA	CMA	CMA	CMA	CMA	CMA	CMA	CEA	CMA
Sponsorship	Novo Nordisk	Novo Nordisk	Baxter	Novo Nordisk	Novo Nordisk	Novo Nordisk	Novo Nordisk	Baxter	Novo Nordisk
Study design
Country	UK	UK	USA	USA	Turkey	USA	Brazil	USA	South Korea
Model	Decision tree	Decision tree	Decision tree	Decision tree	Decision tree	Decision tree	Decision tree	NA	Decision tree
Perspective	National Health Service	National Health Service	Third-party payer	Third-party payer	Reimbursement institutions	Third-party payer	National Health Service	Third-party payer	Reimbursement authorities
Data source	Literature and panelists’ evaluation	Literature and panelists’ evaluation	Literature	Literature and panelists’ evaluation	Retrospective study, literature and panelists’ evaluation	Literature	Observational (rFVIIa) and retrospective (aPCC) study	Prospective study (Base on FENOC11)	Observational (rFVIIa) and retrospective (aPCC) study
Study setting	Home	HCC	Home	Home	Home or HCC	Home	Home or HCC	Home	Home or HCC
Assumptions
Efficacy
aPCC	79%	79% (2nd line: 62%)	78%	75% (1st line)	79.0%	78%	56.7%	NA	63.6%
rFVIIa	92%	92%	87.5%	90% (1st line)	89.3%	92%	100%	NA	92.3%
1st-line dose
aPCC (IU/kg)	75 × 3 = 225	75 × 3 = 225	75 × 3 = 225	75	167*	75 × 2.4 = 180	260*	85	168*
rFVIIa (μg/kg)	90 × 2.3 = 207	90 × 2.3 = 207	90 × 4 = 360	270	204*	90 × 2.3 = 207	190*	105 × 2 = 210	136*
2^nd -ine dose
aPCC
(a) Cont’ or aug’	228–287	480	NA	120 (91%)	NR	180	NR	NA	NR
(b) Switch	NA	NA	NA	NA	NR	NA	NA	NA	NR
rFVIIa
(a) Cont’ or aug’	235–844	567	NA	270 (73%)	NR	207	NR	NA	NR
(b) Switch	844	NA	NA	NA	NR	207	NA	NA	NR
3rd-line dose	Vary	Vary	NA	NA	NR	vary	NR	NA	NR
Drug cost†
aPCC (per IU)	NR	NR	$2.02	$1.93	$1.31	$2.01	$0.73	$2.01	$1.67
rFVIIa (per μg)	NR	NR	$1.91	$2.08	$1.08	$1.84	$0.83	$1.77	$1.63
Body weight (kg)	NR	NR	70	NR	49	70	52.6	NR	49.8
Rebleed rate	15%	15%	NA	NA	15%	15%	15%	NA	rFVIIa: 16.1% aPCC: 12%
Results
Total direct cost†
aPCC	$35,068	$49,010	$25,078	$31,127	$16,319	$36,938–38,453	$16,147	$11,485	$21,631
rFVIIa	$30,995	$28,241	$49,391	$49,507	$11,857	$33,581	$9,078	$25,490	$14,725
Sensitivity analysis	aPCC is cost-saving if aPCC efficacy is 90% or rFVIIa efficacy is lower than 90%	aPCC is cost-saving if the rFVIIa efficacy is below 50% or rFVIIa is given 5 doses of 90 µg/kg	aPCC is the less expensive 99% of the time	Insensitive to changes in drug cost, dosing, and efficacy	rFVIIa is cost-saving if rFVIIa efficacy is >89%	aPCC is cost-saving if rFVIIa efficacy is lower than 82% or rFVIIa unit price is increased by 16%	aPCC is cost-saving if aPCC efficacy is 80% or rFVIIa efficacy is lower than 66%	Sensitive to unit price	Insensitive to changes in efficacy, dose and rebleed rate
Major limitation	Efficacy and dosing assumptions are based non-controlled single armed studies	Efficacy and dosing assumptions are based non-controlled single armed studies	Efficacy and dosing assumptions are based non-controlled single armed studies; Only first line of treatment was considered	Efficacy and dosing assumptions are based non-controlled single armed studies; Dosing for rFVIIa is higher than what reported in other studies; aPCC unit price was not discounted	Data were obtained from a retrospective study with small sample size	Efficacy and dosing assumptions are based non-controlled single armed studies	Data were obtained prospectively for rFVIIa and retrospectively for aPCC in two different time periods	Only first line of treatment was considered; The dosing of rFVIIa is different from what reported in other studies.	Data were obtained prospectively for rFVIIa and retrospectively for aPCC in two different time periods

CMA, cost minimization analysis; CEA, cost effectiveness analysis; NA, not applicable; NR, not reported; cont’, Continue; aug’, augmentation; HCC, hemophilia comprehensive center.

†Costs are adjusted to 2010 US dollar using Purchasing Power Parity (PPP) conversion rate and Consumer Price Index (CPI).The original costs are available upon request from the authors.

*The units are the mean treatment units per body weight by averaging all bleeding episodes treated with each agent.

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