Cost–utility analysis of prophylaxis versus treatment on demand in severe hemophilia A

Figures & data

Figure 1 Structure of the model. The rectangles represent the possible disease states and the arrows indicate the possible transitions between those states.⁶

Figure 2 The four different scenarios simulated in the model.

Notes: *Regimen implemented in Scandinavian countries (Sweden) but not in Italy; **Introduced to represent a treatment strategy currently used in clinical practice in Italy.

Table 1 Input data used to run the Italian model

	On demand	Hybrid regimen	Primary prophylaxis	Secondary prophylaxis	References
Start age (years) secondary prophyl.				2	*
End age (years) primary prophyl.		18			*
No. of patients treated	100	100	100	100	**
Discount rate	6.00%	6.00%	6.00%	6.00%	^Citation7
Annual probability of requiring major surgery	2.30%	0.08%–2.30%	0.08%	0.70%	^Citation7; *; ^Citation7
Dose	40 UI kg	30–35 UI kg	30 UI kg	30 UI kg	^Citation13; *; *
Times per week	1.5		2.5	2.5	*; *; *
No. of weeks	Per bleeding		46	46	*; *; *
Utility			= 1.03 (95% CI 1.00 to 1.06) + (−0.003 × age)		^Citation7
	= 0.84 (95% CI 0.74 to 0.94) + (−0.006 (95% CI −0.008 to −0.004) × age)				^Citation7
Require major surgery	0.190	0.190	0.190	0.190	^Citation7
Bleeding frequency
Adolescents	33.7	2.5–33.7	2.5	33.7–2.5	^Citation16
Adults	36.9	5.4–36.9	5.4	36.9–5.4	^Citation16
Costs
Recombinant plasma/albumin-free	€0.68	€0.68	€0.68	€0.68
ReFacto® (Pfizer) (IU)
Hospitalizations for bleedings or examinations (unit cost)	€4,246	€4,246	€4,246	€4,246	4
Hospitalizations for major surgery (unit cost)	€8,582	€8,582	€8,582	€8,582	4

Notes:

* “Angelo Bonomi Hemophilia and Thrombosis Center”, Milan, Italy

** Assumption.

Figure 3 Sensitivity analysis: primary prophylaxis versus treatment on demand.

Figure 4 Sensitivity analysis: hybrid regimen versus treatment on demand.

Figure 5 Sensitivity analysis: secondary prophylaxis versus treatment on demand.

Table 2 Total costs and incremental cost-effectiveness ratios (ICERs) for phrophylaxis vs treatment on demand in individuals with severe hemophilia A

	On demand	Hybrid regimen	Primary prophylaxis	Secondary prophylaxis
Costs for 100 patients followed up for 70 ys
Recombinant plasma/albumin-free	€87,426,642	€129,600,063	€166,168,643	€164,440,652
ReFacto® (Pfizer) (IU)
Effectiveness	4,137	4,491	6,094	6,051
ICER (vs. on demand)		119,134	40,236	40,229

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