Haemophilia A is a sex-linked recessive genetic disorder associated with haemorrhagic diathesis due to reduced plasma activity of coagulation factor VIII, i.e., below 50% of the normal value (< 0.5 IU/ ml). The treatment of haemophilia A-Inhibitor patients is bidirectional. Major issues with treatment are inhibitor eradication and control of haemorrhage. The aim of the analysis was to evaluate costs and effects of the use of aPCC and rFVIIa in haemophilia A-inhibitor patients in on-demand treatment, perioperative prophylaxis and long-term prophylaxis.
Materials and methods: The cost analysis was performed from the payer's perspective for all treatment schemes. Dosage and duration of treatment were obtained from a systematic review of clinical trials, Summary of Product Characteristics and clinical practice guidelines. Analysis was conducted in accordance with the AHTAPol (Agency for Health Technology Assessment in Poland) guidelines.
Results: The use of aPCC in on-demand treatment of children and adults during one day or one episode of bleeding (irrespective of the intensity of bleeding: from mild to severe) reduced the payer's expenditures in comparison to the use of rFVIIa. The use of aPCC in perioperative prophylaxis of children and adults during minor and major surgical interventions and implantations of central venous access devices was associated with savings for the payer in comparison to the use of rFVIIa, irrespectively of dosage of both drugs. Break even point analysis showed that the use of aPCC in long-term prophylaxis may be less expensive than the use of aPCC in on-demand treatment if bleedings last for 2.5 days or more. Sensitivity analysis showed that assumptions concerning body weight of patients significantly influence expenses of the public payer. However, the use of aPCC was associated with lower costs than the use of rFVIIa, irrespectively of the patients' body weight.
Conclusions: In order to demonstrate the actual size of expenditures on treatment of haemophilia A-Inhibitor patients, collection of data concerning real clinical practice in Poland is required.