Treatment patterns and cost‐of‐illness of severe haemophilia in patients with inhibitors in Germany

Abstract

To evaluate current treatment patterns and resource utilization as well as related cost in the management of severe haemophilia patients with inhibitors in Germany, a cost-of-illness study was conducted. Generally, data were generated by structured literature search. Missing data were collected by expert interviews. All data were validated by a panel of German experts in haemophilia care. In Germany, immune tolerance therapy (ITT) is first-line therapy in inhibitor management for children in the initial year after inhibitor development, particularly for high responders (HR). In adult HR patients ITT is applied but to a remarkably lower extent than in children. To treat bleeding episodes, factor VIII (FVIII) is first-line therapy in low responders (LR). For paediatric HR patients, bleeds are mainly treated with recombinant FVIIa (rFVIIa). In adult HR patients, activated prothrombin complex concentrate (aPCC) and rFVIIa are more equally distributed as treatment options. Treatment costs were calculated for paediatric patients (15 kg) and adult patients (75 kg) from third party payers' perspective. Cost for ITT ranges from Euro 70,290 (2 months; LR) to Euro 3 812,400 (24 months; with aPCC; HR) in a paediatric patient. For an adult patient ITT cost ranges from Euro 287,500 (6 months; LR) to Euro 17,253,000 (36 months; HR). For on average 12.5 acute bleeds, average annual treatment costs amount to Euro 77,000 for a child and Euro 354,000 for an adult. Assessing the results it has been taken into consideration that ITT can last longer and annual number of bleeds can be extremely higher than on average 12.5 episodes. This indicates more health care resource consumption in some patients.

Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

“…Both France and Italy found the total cost of treating patients in the prophylaxis group was significantly higher (around 3.5 times, 3.7-4.1 times for direct costs) than in the OD group, while in Germany prophylaxis group costs were lower than the OD group. Hospitalisation costs were highest in the French OD group (64% of direct There appears to be no significant variation in hospitalisation costs between type A and B haemophilia (91), but annual treatment costs may vary with age, as German evidence shows paediatric patients, both with and without inhibitors, cost much less to treat than adults (by more than 4 times in both groups) (92).…”

Optimizing management of immune tolerance induction in patients with severe haemophilia A and inhibitors: towards evidence‐based approaches

“…Annual costs of ITI were calculated to be almost 15-fold higher than on-demand treatment in inhibitor patients (Auerswald et al, 2004). A relevant impact of ITI duration on costs, reflecting the patient prognostic profile, has been highlighted (Aledort et al, 2000).…”

Burden of illness: direct and indirect costs among persons with hemophilia A in the United States

“…In a 2004 study in Norway and Sweden, clotting factor consumption in prophylaxis was 2-3-times higher than that in episodic treatment, accounting for 94% vs 74% of total costs, respectively 8 . In addition, 25-30% of patients with severe hemophilia A and 4-6% with severe hemophilia B may develop antibodies (inhibitors) and require higher doses of clotting factors or other treatments 9 , which result in even higher medical costs 10,11 . Affected individuals also incur considerable indirect costs, including lost productivity, costs for caregivers' unpaid time and hemophilic individuals' disability 12 .…”