Abstract
Objective The aim of this study was to assess the consumption of anti-haemophilic drugs by adults and children with severe haemophilia A or B (residual activity of FVIII or FIX ≤2%) and to quantify the average direct medical costs. Method A retrospective multicentre cost-of-illness study from the perspective of French national health insurance system. The costs include only the use of clotting factors. Main outcome measure Consumption was expressed in UI/kg/year and costs in euros/kg/year. Results From January 1, 2001 to December 31, 2002, data from 81 adults and 30 children with severe haemophilia A (n = 92) or B (n = 19) and included in the “SNH” were collected and analysed. A coagulation factor inhibitor was present in 10 patients (9%). Four of them were high responders. Mean age and body weight were respectively 28 ± 17 years and 58 ± 24 kg. Except for one adult patient, all (99%) had outpatient treatment, 44 patients (40%) were hospitalized and treated by recombinant or/and plasma-derived FVIII or FIX or/and rFVIIa. Overall median annual consumption of anti-haemophilic drugs per patient was estimated at 1,333 UI/kg, with a median cost-of-illness of 1,156 euros/kg. Patients with severe haemophilia B consumed more than patients with severe haemophilia A, though not significantly (P = 0.096), with a median of 2,167 vs. 1,100 UI/kg/year and a median cost of 1,760 vs. 917 euros/kg/year (P = 0.13). Children consumed respectively more than adults (P = 0.008), with a median of 3,204 vs. 1,106 UI/kg/year and a median cost of 2,614 vs. 913 euros/kg/year (P = 0.012). The median cost for patients with an inhibitor was 3,291 euros/kg/year, approximately threefold higher than that of patients without an inhibitor (926 euros/kg/year) (P = 0.022). Conclusion It suggests a higher consumption and cost of anti-haemophilic drugs among children when compared to adults. Haemophilia B patients did not consume significantly more than haemophilia A patients, whereas the consumption and cost for patients with or without inhibitors differed significantly.
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References
Macik BG. Treatment of factor VIII inhibitors: products and strategies. Semin Thromb Haemost 1993;19(1):13–24
DiMichele DM. Inhibitors in haemophilia: a primer. Haemophilia 2000; 6(1):38–40
Sultan Y. The French Hemophilia Study Group. Prevalence of inhibitors in a population of 3,435 hemophilia patients in France. Thromb Haemost 1992;67:600–2
Molho P, Rolland N, Lebrun T, Dirat G, Courpied JP, Croughs T, et al. Epidemiological survey of the orthopaedic status of severe haemophilia A and B patients in France. Haemophilia 2000;6(1):23–32
Jasso-mosqueda JG, Omnès LF, Gomez E, et al. Description of the management of patients suffering from type A haemophilia [Description de la prise en charge de l’hémophilie sévère de type A par le programme de médicalisation des Systèmes d’information : intérêts et limites]. J Econ Med 2000;18:323–30
D’Alche-Gautier MJ, Gautier P, Stieltjes N, et al. Economic study of clotting factor therapy in haemophilia [Coût du traitement substitutif des hémophiles hospitalisés]. J Econ Med 2001;19:157–66
Schramm W, Royal S, Kroner B, Berntorp E, Giangrande P, Ludlam C, et al. Clinical outcomes and resource utilization associated with haemophilia care in Europe. Haemophilia 2002;8(1):33–43
Auerswald G, Von Depka Prondzinski M, Ehlken B, Kreuz W, Kurnik K, Lenk H, et al. Treatment patterns and cost-of-illness of severe haemophilia in patients with inhibitors in Germany. Haemophilia 2004;10:499–508
Chang H, Sher GG, Blanchette VS, Teitel JM. The impact on the cost of clotting factor replacement therapy in haemophilia A in Canada. Haemophilia 1999;5(4):247–52
Rivard GE, Vick S. Factor VIII inhibitor treatment. Economics of inhibitor treatment in Canada. Semin Hematol 1994;31(4):41–3
Bohn RL, Aledort LM, Putnam KG, Ewenstein BM, Mogun H, Avorn J. The economic impact of factor VIII inhibitors in patients with haemophilia. Haemophilia 2004;10(1):63–8
Gautier P, D’Alche-Gautier MJ, Coatmelec B, Marques-Verdier A, Bertrand MA, Dieval J, et al. Cost related to replacement therapy during hospitalization in haemophiliacs with or without inhibitors: experience of six French haemophilia centres. Haemophilia 2002;8(5):674–9
Goudemand J. Hemophilia. Treatment of patients with inhibitors: cost issues. Haemophilia 1999;5(6):397–401
Ehrenforth S, Kreuz W, Scharrer I, Linde R, Funk M, Güngör T, et al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet 1992;339:594–8
Calvez T, Biou M, Costagliola, Jullien AM, Laurian Y, Rossi F, et al. The French haemophilia cohort: rationale and organization of a long-term national pharmacosurveillance system. Haemophilia 2001;7:82–8
Gill FM. The natural history of factor VIII inhibitors in patients with haemophilia A. Prog Clin Biol Res 1984;150:19–29
Shapiro A. Inhibitor treatment: state of the art. Semin Hematol 2001;38:26–34
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We are indebted to Bayer Laboratory and Biotech Division for logistic and organisational assistance. We also thank Ms Pamela Albert for English assistance in the correction of the manuscript.
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Nerich, V., Tissot, E., Faradji, A. et al. Cost-of-illness study of severe haemophilia A and B in five French haemophilia treatment centres. Pharm World Sci 30, 287–292 (2008). https://doi.org/10.1007/s11096-007-9181-4
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DOI: https://doi.org/10.1007/s11096-007-9181-4