Cost-minimization analysis of recombinant factor VIII Fc versus emicizumab for treating patients with hemophilia A without inhibitors in Europe

Figures & data

Table 1. Model and scenario analyses inputs.

Input	Value	Source
Population size	100 Patients	Assumption
Time horizon	5 Years	–
Country	UK, France, Italy, Spain, and Germany	–
Mean (SD) bodyweight male adolescents/adults (≥12 years), kg	73.51 (14.95)	A-LONG trial^14
Mean (SD) bodyweight male children (<12 years), kg	24.99 (11.85)	Kids A-LONG trial^16
Wastage	As recommended by manufacturer	Emicizumab dosing calculator^24
rFVIIIFc dose – prophylaxis (adolescents/adults)	85.4 IU/kg/week	A-LONG trial^14^a
rFVIIIFc dose – prophylaxis (children)	88.11 IU/kg/week	Kids A-LONG trial^16
Emicizumab dose – prophylaxis (loading)	3 mg/kg Q1W	Summary of Product Characteristics^22
Emicizumab dose – prophylaxis (maintenance)	1.5 mg/kg Q1W	HAVEN 3^25
FVIII dose to treat breakthrough bleeds	44.53 IU/kg	A-LONG trial^14
ABR rFVIIIFc	2.9	A-LONG trial^14 (individualized prophylaxis)
ABR emicizumab	1.56	HAVEN 3^25 (1.5 mg/kg QW)
Proportion of emicizumab-treated patients without bleeds	55.8%	HAVEN 3^25 (1.5 mg/kg QW)
Proportion of emicizumab-treated patients requiring dose up-titration	0%	HAVEN 3^25
rFVIIIFc list price (€ per IU)	UK: 0.89 France: 0.65 Italy: 0.65 Spain: 0.59 Germany: 0.53	SOBI data
Emicizumab list price (€ per mg)	UK: 80.51 France: 77.13 Italy: 55.07 Spain: 76.70 Germany: 64.66	SOBI data
Octocog alfa list price (per IU)	UK: €0.84 France: €0.65 Italy: €0.63 Spain: €0.56 Germany: €1.08	UK: MIMS data Other countries: Sobi data

^aMean dose from individualized prophylaxis arm in A-LONG.

Abbreviations: ABR, annualized bleed rate; MIMS, Monthly Index of Medical Specialities; NHS, National Health Service; Q1W, once weekly; SD, standard deviation.

Figure 1. Five-year costs associated with using rFVIIIFc and emicizumab for prophylactic treatment of 100 people with hemophilia A (base-case analysis: recommended wastage; emicizumab 1.5 mg/kg once weekly). (a) Adolescents/adults (≥12 years). (b) Children (<12 years). Abbreviation: rFVIIIFc, recombinant factor VIII Fc.

Figure 2. Scenario analyses (UK): 5-year costs associated with using rFVIIIFc and emicizumab for prophylactic treatment of 100 people with hemophilia A, according to (a) Reduced dosing frequency for emicizumab, (b) Emicizumab potential maximum wastage, and (c) Maximal emicizumab wastage according to bodyweight. (a) Base-case dosing interval for emicizumab (1.5 mg every week) included for comparative purposes. (b) Elocta dose set at 88.11 IU/kg/week. Abbreviations: QW, once weekly; Q2W, every 2 weeks; Q4W, every 4 weeks; rFVIIIFc, Recombinant factor VIII Fc.

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Data availability statement

The data that support the findings of this study are available from the corresponding author [GC], upon reasonable request.