Introduction
Hemophilia A (HA) poses a high economic burden on the National Health System (NHS). This is especially relevant for those patients who develop inhibitors to standard factor VIII replacement therapies.
To establish health policies, it is of importance to have information on the annual cost of managing this group of patients.
Objective
The objective of this study is to estimate the average annual pharmacological cost of a patient with HA and inhibitors, from the perspective of the NHS.
Methods
An economic model to estimate the annual pharmaceutical cost associated with bypassing agent therapy (activated prothrombin complex concentrate-aPCC and activated recombinant factor VII-rFVIIa) in HA patients with inhibitor was developed.
The population was stratified by age: pediatric patients (<14 years) and adults (≥14 years), and therapeutic strategy: prophylaxis or on-demand treatment of the bleeding episodes.
Patients’ characteristics and the location, severity and duration of bleeding episodes were obtained from the literature.
The pharmaceutical cost per bleeding was calculated considering the annual number of bleedings according to age group and therapeutic strategy. The unitary cost of the alternatives was expressed as ex-factory price, including national mandatory deduction.
Results
Average annual cost per HA patient with inhibitor in Spain was estimated at €430,227, with the prophylactic strategy with aPCC entailing an average cost of €636,905 per adult patient and €264,819 per paediatric patient.
Annual cost of on-demand treatment was €564,917/adult patient and €203,970/paediatric patient, with considerable variations between treatment with aPCC or rFVIIa.