Health Technology Assessment di AdvateTM (octocog alfa) per la profilassi del sanguinamento nei pazienti con Emofilia A. Ann Ig 2013; 25 (Suppl. 1): 1-7

This study aimed at assessing the global value of AdvateTM, antihaemophilic recombinant factor for bleeding prevention and control in Haemophilia A. Epidemiological, clinical, organizational and social and ethical data related to available Haemophilia treatments were considered. Scientific literature reviews were conducted through all main electronic databases. A cost-effectiveness analysis (CEA) from the perspective of the National Health Service (NHS) was performed in order to calculate the incremental cost-effectiveness ratio (ICER) comparing the use of octocog alfa in primary prophylaxis vs on-demand treatment. In the world, patients with Haemophilia A are approximately 110,000 (about 3700 in Italy). Prophylaxis,compared to on-demand treatment, is proven to be more effective for prevention of bleedings and related complications, but also moderately more expensive (ICER= €35,036/QALY). The introduction of totally protein-free full length recombinant FVIII concentrates (AdvateTM), eliminated risks of blood-borne pathogens transmission. This induced a broader adoption of prophylaxis improving efficacy of treatment and patients’ Quality of Life (QoL). Also, since Baxter, AdvatÈs manufacturer and distributor, offers collateral free of charge services to patients (home delivery, home rehabilitation assistance, home nursing, telemedicine), the HTA looks at how all these services generate savings for the healthcare authorities while increasing the level of assistance. The evidence provided by HTA should be taken into account by decision-makers to improve the allocation of resources and rationalize health choices.