Haemophilia A: Pharmacoeconomic Review of Prophylaxis Treatment versus On-Demand

Table 1

Characteristics of the included studies.

Reference	Type of analysis	Alternative	Country/perspective	Patients/time horizon	Effectiveness measurement/cost measurement	Results
Miners et al., 2002 [14]	Cost-utility analysis	Primary prophylaxis versus on-demand with clotting factors	UK/societal	Hypothetical cohort of 100; patients with severe haemophilia A o B/severe Von Willebrand disease; Time horizon: lifetime	QALYs/direct medical costs and indirect costs	QALY: primary prophylaxis; 55.89 versus on-demand 41.10; ICER: (€59.315^a)/QALY; Primary prophylaxis is more cost-effective than on-demand therapy
Lippert et al., 2005 [17]	Cost-utility analysis	Secondary prophylaxis versus on demand	Germany, Sweden, UK, and The Netherlands/third party payer	Patients with severe haemophilia A and B without inhibitor (age 14 years); Time horizon: one year	QALYs and avoided bleeding episodes/direct medical costs	ICER: patients ≤ 30 years old and HIV positive €1.2 million/QALY (Germany); €1.72 million/QALY (UK); Patients ≤ 30 years old and HIV negative €2.21 million/QALY (Germany); €3.10 million/QALY (UK); Patients > 30 years old and HIV negative €4.77 million/QALY (Germany) €5.7 million/QALY (Sweden, UK); Prophylaxis is more effective than on-demand treatment, but it causes higher costs
Risebrough et al., 2008 [16]	Cost-utility analysis	EscDose versus on-demand; Primary prophylaxis versus EscDose	Canada/societal	Individuals with severe haemophilia A, between 1 and 6 years old; Time horizon: 5 years	QALYs and joint bleeding events avoided/direct medical costs and indirect costs (work days lost by parents)	EscDose versus on demand: significant reduction of bleeding (bleeding episodes decreased by 52 joint-bleeds when EscDose is compared to on-demand therapy); ICER: Can$542.938 (€379.134^b)/QALY.; EscDose versusprimary prophylaxis; ICER: >Can$1.000.000 (€698.300^b)/QALY.; Prophylaxis can improve the quality of life of patients with haemophilia, though with high costs
Miners, 2009 [15]	Cost-utility analysis	Primary prophylaxis versus on-demand with FVIII	UK/NHS	Hypothetical cohort of patients with severe haemophilia type A; Time horizon: lifetime	QALY/direct medical costs	QALY: Primary prophylaxis 19.58 versus on-demand 13.95.; Primary prophylaxis versus on-demand; ICER: (€48.469^a)/QALY; Primary prophylaxis is cost-effective compared with on demand therapy.
Colombo et al., 2011 [18]	Cost-utility analysis	On-demand treatment versus (1) primary prophylaxis; (2) secondary prophylaxis; (3) hybrid regimen (primary prophylaxis followed by on-demand)	Italy/NHS	Hypothetical cohort of patients with severe haemophilia A; Time horizon: lifetime	QALY/direct medical costs	ICER on-demand versus; Primary prophylaxis: €40.236/QALY; Secondary prophylaxis: €40.229/QALY; Hybrid regimen: €119.134/QALY.; Primary and secondary prophylaxes are more cost-effective than the hybrid regimen.

Exchange rate /€1.2758, updated to November 4, 2014. ^bExchange rate Can$ 1/€0.6983, updated to November 4, 2014.