Cost Study Of Immune Thrombocytopenia (ITP) Management From The French Hospital Perspective

• Published in: Value in health 2017-10, Vol.20 (9), p.A551
• Main Authors: Cariou, C, Affinito, S, Lafon, T, Blein, C, Duteil, E, Sion, M, Mahieu, N, Duco, J, Leclerc-Teffahi, S, Cheze, S
• Format: Article
• Language: English
• Subjects: Bleeding; Cohort analysis; Cost analysis; Dosage; Drug prices; Efficacy; Health care expenditures; Hemophilia; Idiopathic thrombocytopenic purpura; Laparoscopy; Medical diagnosis; Medical treatment; Patients; Pharmacokinetics; Prices; Prophylaxis; Sensitivity analysis; Thrombocytopenia; Uncertainty; Variability
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2017.08.864

OBJECTIVES: To demonstrate the impact of a novel prophylactic treatment on the uncertainty of drug treatment costs in patients with Haemophilia A with inhibitors. For patients with inhibitors, the only haemostatic options currently available are bypassing agents (BPA).The haemostatic effect of BPA in PWHA with inhibitors is suboptimal, leading to a higher number of bleeds compared to patients without inhibitors. METHODS: Average per person annual BPA drug treatment costs were estimated using four sources of data. BPA on demand dosages and bleeding rates were taken from HAVEN 1, a pivotal Phase III study designed to evaluate the efficacy, safety, and pharmacokinetics of once weekly emicizumab prophylaxis compared with no prophylaxis. BPA prophylaxis dosing was obtained from a non-interven-tional study (NIS) BH29768. Average weights of patients were obtained from the UK National Haemophilia Database (NHD). List prices were used for drug costs. Deterministic sensitivity analyses were conducted to determine the range of BPA costs. RESULTS: Estimated annual per patient BPA costs in the 40+ age groups were £1,329,514 for prophylactic use and for on demand £433,065. Variability in annual bleed rates and BPA dosing resulted in a range in BPA costs of +/- 26.9% for prophylactic and +/- 67.7% for on demand bypassing agents. The reduction in the number of bleeds with emicizumab reduces the uncertainty in annual bypassing agent drug costs to a range of just +/- 4%. CONCLUSIONS: An effective, widely used and fixed dose prophylactic treatment for PWHA patients with inhibitors has the potential to greatly reduce the uncertainty in predicting drug treatment costs which in the UK account for over 95% of the total treatment costs. This should aid in budget planning when, for patients who experience a large number of bleeds, annual BPA treatment costs can easily broach £500,000 with on demand or over £1,250,00 for prophylaxis.