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

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Abstract
Summary. 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 €70 290 (2 months; LR) to €3 812 400 (24 months; with aPCC; HR) in a paediatric patient. For an adult patient ITT cost ranges from €287 500 (6 months; LR) to €17 253 000 (36 months; HR). For on average 12.5 acute bleeds, average annual treatment costs amount to €77 000 for a child and €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.