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Health economics

Health economics of factor VIII products

In the era of budget constraints in which we live, it is necessary to make decisions on how to allocate the limited available resources, and to establish priorities in the management of haemophilia patients. This article looks at health economic evidence

Luciana Scalone CHARTA Foundation, Milan, Italy Research Centre on Public Health (CESP), University of Milan-Bicocca, Monza, Italy Lucia S D’Angiolella CHARTA Foundation, Milan, Italy Lorenzo G Mantovani CHARTA Foundation, Milan, Italy Department of Clinical Medicine and Surgery, University Federico II of Naples, Naples, Italy

Over the years, management of haemophilia has allowed the sensitive prolongation of patients’ life expectancies, and is now mainly focused on reducing the occurrence and severity of orthopaedic impairment and on improving patients’ quality of life (QoL).1–3

However, the complex nature of the disease and related consequences, such as the impairment of orthopaedic function and the several aspects related with replacement treatment (for example, effectiveness, risks and high cost), contribute to complicating the decision- making process4,5

and to absorbing huge 30

amounts of socio-economic and human resources. The situation becomes even more complex when haemophilic patients develop inhibitors against the replacement treatment with coagulation factor concentrates, which compromises the mainstay of treatment.6,7 Furthermore, in the era of budget constraints in which we live, it is necessary to make decisions on how to

allocate the limited available resources, and to establish priorities. In particular, replacement treatment with coagulation factor concentrates is a main cause of increase of healthcare costs attributable to haemophilia, contributing up to 95–99% of total costs, especially when used for prophylaxis8,9

or to manage patients with

inhibitors, who need higher dosages of factor FVIII/IX or bypassing agents.7 Although replacement treatments have been largely adopted in at least high- income countries, the high cost of coagulation factor concentrates could actually constitute a barrier to the widespread acceptance of treatment, especially for the prophylaxis regimen.5,10 In this regard, the ESCHQoL Study,10


which 21 European countries and approximately 1400 haemophilia A and B patients were involved, showed interesting trends of haemophilia care provision. The countries participating in the project were grouped into three regions, according to per capita amount of clotting factor used. Both paediatric and adult patients in region 3 (where < 2 IU per capita of clotting factor were used) had median numbers of three and eight joint bleeds, respectively, and worse joint scores, compared to those in region 1 (>5UI per capita used), where zero and one bleed, respectively, were registered. Prophylactic treatment was used in only 31% of children and 9% of adults with severe haemophilia in region 3, compared to 94% and 54%, respectively, in region 1.

Costs and outcomes in haemophilia The high costs attributable to haemophilia and its management have stimulated increasing attention and have promoted the conducting of several studies in the various healthcare systems, with the aim of estimating the socio- economic impact and cost effectiveness of the available treatment options for haemophilia patients with different characteristics and needs. For instance, among the research studies conducted in recent years, a cost-of-illness study carried out in Italy, COCHE,3


the socio-economic impact and QoL among adult moderate and severe patients with haemophilia A and B, without inhibitors. Eighty-six percent of the 232 patients (mean age = 37 years) enrolled in the study had haemophilia A, 72% of whom were severely affected. At baseline, 32.6% of the patients were treated prophylactically, while the others were treated on-demand. Overall, the treatment with coagulation factor concentrates cost €8800 per patient per month. However, when stratifying according to treatment regimen, patients on demand cost on

The results obtained from this first large European study show a clear gradient regarding the joint scores and per capita dose of clotting factor used in the different regions of residence, making residence in a low-dose region a prominent risk factor for arthropathy.

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