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Health economics

average €4300 per patient per month, while those on prophylaxis cost €16,800 per patient per month. On average, these patients showed impaired levels of physical QoL, probably due to orthopaedic impairment, while the mental component of wellbeing was relatively good when compared with the general male population. The main reason that patients do not appear to be psychologically affected by their condition could be the consciousness of availability of effective treatments.3 Nowadays, the prevention of arthropathy is a major goal of haemophilia treatment, which is expected to be attained with prophylactic treatment. However, while some data exist on the higher costs generated by a prophylactic regimen, at least in the short run, little is still known about the overall efficiency of prophylaxis regimen in the long run. This takes into consideration clinical effectiveness and wellbeing, as well as cost of treatment and difficulties related with coping with the administration of drug (for example, venous access, complications related with indwelling catheter and compliance). The ESPRIT randomised clinical trial,11 targeting children with severe haemophilia A, who were observed for a median of seven years, has shown that prophylaxis is effective in preventing bleeds and artrhopathy, especially when started at an early age. An expenditure of €2500 per patient per month was estimated to maintain intact joints of a child with haemophilia.

Another main issue to be considered in haemophilia is related to the development of inhibitors against factor concentrates. As inhibitors reduce the effectiveness of replacement therapy, despite the success of treatment with by-passing agents, haemorrhages in inhibitor patients can become particularly frequent and difficult to control, leading to a higher risk of progressive joint disease and severe mobility impairment. Immune tolerance induction (ITI) is generally recognised as the most preferred treatment option to be used for the eradication of inhibitors, although in several patients ITI is actually not successful.12

Furthermore, little is

known about the long-term benefits and costs attributable to ITI. In the multinational observational ITER Study,6 seventy-one adult and paediatric haemophilia A patients from 11 European haemophilia care centres underwent ITI treatment using factor VIII. Before

inhibitor diagnosis, the mean treatment cost was €670.20 per patient per month, while it increased to €3188 per patient per month after inhibitors diagnosis and until ITI start. ITI was successful in 84.5% of patients at a mean cost of €60,000 per patient per month during ITI, plus €13,000 per patient per month for one year later.

When eradication of inhibitors is not possible, patients are treated with higher amounts of factor VIII or IX, or with bypassing agents.7,12

An average total

healthcare cost of €18,000 per patient per month was estimated for the treatment of patients with high titer of inhibitors,7 comparable with the cost of prophylaxis

aspects of haemophilia and related issues, nowadays, making decisions in haemophilia care is often difficult, as it actually involves a complex interaction of factors (treatment effects and risks, perceived wellbeing, costs) and of subjects (physicians, patients, and also budget holders) who play different roles in the healthcare system, each carrying his own set of needs (for example, patients’ clinical status, treatment costs experiences, expectations and preferences).4,5,13,14

Together with knowledge on effects and costs, information about preferences of the different subjects involved in the decision-making process can help to

“The management of haemophilia and related consequences should be considered as a long- term investment, requiring the conduct of complex decisions for the optimisation of benefits”

treatment in patients without inhibitors.3 In inhibitor patients, bypassing agents (aPCC and recombinant factor VIIa) contributed to more than 60% of costs, while 35% of costs were attributable to the use of plasma-derived or recombinant factor VIII. Similarly to those without inhibitors, patients with inhibitors reported compromised levels of physical QoL but relatively good levels of mental wellbeing,3

suggesting that, not only do

they receive effective treatments, but also these treatments are satisfactory and generally well accepted by the patients. Despite the availability of several research works conducted on different

optimise benefits and efficiency of the different treatments and strategies. For instance, in the short run, understanding preference of patients can help health professionals optimise disease management by identifying their main concerns, hence improving adherence and satisfaction with treatment. In the long run, knowledge on preferences can guide the development of future drugs to help fulfil patients’ needs and expectations. In recent years, two multicentre observational studies have been carried out to assess and compare preferences for the characteristics of treatment of patients with haemophilia A or B.13,14



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