OBJECTIVES: Currently, substitution of clotting factors is the most commonly used option for patients affected by haemophilia. Factors are well established, but expensive. This research aims to explore the cost of the most widely used clotting factors in Austria and compares these with projected costs of gene therapy.

METHODS: We analysed the most widely used products for the treatment of haemophilia A (HA) and haemophilia B (HB) with standard half-life (SHL) and extended half-life (EHL), respectively. In Austria, most patients are diagnosed at birth, hence a time horizon of 67 years of maintenance therapy was chosen. The average patient weight was assumed to be 72,5 kg. The analysis was performed from the payer perspective restricting it to direct costs. Costs were extracted from Austrian cost catalogues using ex-factory prices.

RESULTS: For HA and HB, annual maintenance costs varied between €88.595 and €313.272 for SHL factors and between €56.414 and €318.052 for EHL products. Without consideration of inflation, costs of additional on-demand factor usage or immune tolerance induction, lifetime costs vary between €3,8 M and €21,3 M per patient in Austria.

CONCLUSIONS: Even though the cost of gene therapy for haemophilia is still unknown, considering cost-effectiveness gene therapy has the potential to offer good value for money to the Austrian healthcare system. It is unlikely that the direct costs of gene therapy will be equal to the lifetime cost of clotting factors, and additional benefits offered by gene therapy need to be taken into account. These include quality of life improvements for patients and caretakers, improved compliance and fewer bleeds due to a durable response. Further research is needed around the projected costs of gene therapy and the wider implications of better haemophilia care to society via a reduction of indirect costs and caregiver burden.