Clinical and economic analysis of different strategies for the treatment of hemophilia A
DOI:
https://doi.org/10.24959/cphj.17.1414Keywords:
hemophilia, inhibitor, NovoSeven, Feiba, pharmacoeconomic analysis, decision treeAbstract
Although hemophilia A is a rare disease, its treatment is costly, and expenses are mainly related to the cost of factor VIII concentrates.
Aim. To evaluate the clinical and economic feasibility of rFVIIa (NovoSeven®) for the treatment of patients with hemophilia A compared to the schemes based on the use of APCC (Feiba).
Matherials and methods. Clinical and economic analysis was conducted by the “total cost of illness” method using modeling by building a “decision tree”. This study identifies only the direct costs of pharmacotherapy. The cost of a vial
of each of the concentrates analyzed meets the declared value as of December 2016.
Results. The analytical model of the “decision tree” was designed to calculate the costs for the outpatient treatment of mild and moderate bleedings. Due to higher clinical efficacy the use of rFVIIa as the first-line therapy saves 32 % to 42 % in the treatment of a patient; in the conditions of the modern Ukrainian pharmaceutical market it is from UAH 170,326.4 to UAH 252,990.4 (with an adapted variant of therapy) and UAH 291,743.1 in the basic variant. When using rFVIIa as the first-line therapy the costs for life appeared to be lower by approximately 10 % than when taking APCC.
Conclusions. The use of NovoSeven® is more feasible economically and allows both to improve outcomes and to spend financial resources more efficiently to treat these patients.
References
The diagnoses and management of factor VIII and IX inhibitors: a guideline from the United Kingdom Haemophilia Centre
Doctors Organisation / C. Hay, S. Brown, P. Collins et al. // Br. J. Haematol. – 2006. – Vol. 133, Issue 6. – P. 591–605. doi:
1111/j.1365-2141.2006.06087.x
Berntorp, E. Importance of rapid bleeding control in haemophilia complicated by inhibitors / E. Berntorp // Haemophilia. –
– Vol. 17, Issue 1. – P. 11–16. doi: 10.1111/j.1365-2516.2010.02337.x
Odayemi, I. Modelling the economic impact of recombinant activated factor VII and activated prothrombin complex concentrate in
the treatment of a mild to moderate bleed in adults with inhibitors to clotting factor VIII and IX at a comprehensive care centre
in the UK / I. Odayemi, J. Guest // J. of Med. Economics. – 2002. – Vol. 5, Issue 1–4. – P. 51–64. doi: 10.3111/200205051064
Cost of care of haemophilia with inhibitors / M. Di Minno, G. Di Minno, M. Di Capua et al. // Haemophilia. – 2010. – Vol. 16,
Issue 1. – P. e190–e201. doi: 10.1111/j.1365-2516.2009.02100.x
Berntorp, E. Differential response to bypassing agents complicates treatment in patients with haemophilia and inhibitors
/ E. Berntorp // Haemophilia. – 2009. – Vol. 15, Issue 1. – P. 3–10. doi: 10.1111/j.1365-2516.2008.01931.x
Sequential therapy with activated prothrombin complex concentrates and recombinant FVIIa in patients with severe haemophilia
and inhibitors: update of our previous experience / J. Schneiderman, E. Rubin, D. Nugent, G. Young // Haemophilia.
– 2007. – Vol. 13, Issue 3. – P. 244–248. doi: 10.1111/j.1365-2516.2007.01451.x
Valentino, L. A. Assessing the benefits of FEIBA prophylaxis in haemophilia patients with inhibitors / L. A. Valentino // Haemophilia.
– 2009. – Vol. 16, Issue 2. – P. 263–271. doi: 10.1111/j.1365-2516.2009.02126.x
Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors
/ B. Konkle, L. Ebbesen, E. Erhardtsen et al. // J. of Thrombosis and Haemostasis. – 2007. – Vol. 5, Issue 9. – P. 1904–1913.
doi: 10.1111/j.1538-7836.2007.02663.x
Randomized comparison of prophylaxis and on-demand regimens with Feiba NF in the treatment of haemophilia A and B
with inhibitors / S. Antunes, S. Tangada, O. Stasyshyn et al. // Haemophilia. – 2013. – Vol. 20, Issue 1. – P. 65–72. doi: 10.1111/
hae.12246
Single 270 μg kg-1 – dose rFVIIa vs. standard 90 μg kg-1 – dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia
patients with inhibitors: a randomized comparison / G. Young, F. Shafer, P. Rojas, S. Seremetis // Haemophilia. – 2008. – Vol. 14,
Issue 2. – P. 287–294. doi: 10.1111/j.1365-2516.2007.01601.x
A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative
(FENOC) Study / J. Astemark, S. Donfield, D. DiMichele et al. // Blood. – 2007. – Vol. 109, Issue 2. – P. 546–551. doi: 10.1182/
blood-2006-04-017988
Knight, C. Systematic review of efficacy of rFVIIa and aPCC treatment for haemophilia patients with inhibitors / C. Knight,
A. Danø, T. Kennedy-Martin // Advances in Therapy. – 2009. – Vol. 26, Issue 1. – P. 68–88. doi: 10.1007/s12325-008-0135-6
Economic modelling of different treatment strategies for haemophilia A with high-responding inhibitors / C. Knight, S. Paisley,
J. Wight, M. Jones // Haemophilia. – 2003. – Vol. 9, Issue 4. – P. 521–540. doi: 10.1046/j.1365-2516.2003.00783.x
Pharmacoeconomic analysis of recombinant factor VIIa versus APCC in the treatment of minor-to-moderate bleeds in hemophilia
patients with inhibitors / A. Joshi, J. Stephens, V. Munro et al. // Current Med. Res. and Opinion. – 2005. – Vol. 22, Issue 1. –
P. 23–31. doi: 10.1185/030079906x80224
A cost-evaluation of treatment alternatives for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors
in Brazil / M. Ozelo, P. Villaca, J. De Almeida et al. // Haemophilia. – 2007. – Vol. 13, Issue 5. – P. 462–469. doi:
1111/j.1365-2516.2007.01522.x
A cost evaluation of treatment alternatives in mild-to-moderate bleeding episodes in haemophilia patients with inhibitors
in Turkey / S. Dundar, B. Zulfikar, B. Kavaklik et al. // J. of Med. Economics. – 2005. – Vol. 8, Issue 1–4. – P. 45–54. doi:
3111/200508046054
Pharmacoeconomic evaluation of treatment alternatives for mild to moderate bleeding episodes in patients with haemophilia
with inhibitors in Korea. Abstract from the XXI International Society on Thrombosis and Haemostasis Congress 2007
/ S. Yoo, L. Yong, P. Kyu, Y. Woo // J. of Thrombosis and Haemostasis. – 2007. – Vol. 5. – P. 158.
Economic analysis of recombinant activated factor VII versus plasma-derived activated prothrombin complex concentrate
in mild to moderate bleeds: Haemophilia registry data from the Czech Republic / P. Salaj, M. Penka, P. Smejkal, V. Geierova,
P. Ovesná, P. Brabec et al. // Thrombosis Res. – 2012. – Vol. 129, Issue 5. – P. e233–e237. doi: 10.1016/j.thromres.2012.02.005
Cost analysis of prophylaxis with activated prothrombin complex concentrate vs. on-demand therapy with activated factor
VII in severe haemophilia A patients with inhibitors, in Spain / R. Villarrubia, I. Oyagüez, M. Álvarez-Román et al. // Haemophilia.
– 2015. – Vol. 21, Issue 3. – P. 320–329. doi: 10.1111/hae.12681
Cost-Effectiveness Analysis of Biogeneric Recombinant Activated Factor VII (AryoSeven™) and Activated Prothrombin Complex
Concentrates (FEIBA™) to Treat Hemophilia A Patients with Inhibitors in Iran / M. Golestania, P. Eshghib, H. Rasekh et
al. // Iranian J. of Pharmaceutical Res. – 2016. – Vol. 15. – P. 669–677.
Jo, C. Cost-of-illness studies: concepts, scopes, and methods / C. Jo // Clinical and Molecular Hepatol. – 2014. – Vol. 20, Issue 4. –
P. 327. doi: 10.3350/cmh.2014.20.4.327
Cost-of-illness Studies / E. Akobundu, J. Ju, L. Blatt, C. Mullins // Pharmacoeconomics. – 2006. – Vol. 24, Issue 9. – P. 869–890.
doi: 10.2165/00019053-200624090-00005
Фармакоекономіка : навч. посіб. для студентів ВНЗ / Л. Яковлєва, Н. Бездітко, О. Герасимова та ін. – Вінниця : Нова
книга, 2009. – 208 с.
Briggs, A. Decision Modelling for Health Economic Evaluation / A. Briggs, M. Sculpher, K. Claxton // Handbooks in Health
Economic Evaluation. – Oxford University Press, 2006. – P. 256.
Реєстр оптово-відпускних цін на лікарські засоби [Електронний ресурс]. – 23.12.2016. – Режим доступу до ресурсу :
http://www.moz.gov.ua/ua/portal/register_prices_drugs.
Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study / E. Santagostino,
M. Mancuso, A. Rocino et al. // Brit. J. of Haematol. – 2005. – Vol. 130, Issue 3. – P. 422–427. doi: 10.1111/j.1365-2141.2005.05605.x
Gouw, S. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the
CANAL cohort study / S. Gouw, J. van der Bom, H. van den Berg // Blood. – 2007. – Vol. 109, Issue 11. – P. 4648–4654. doi:
org/10.1182/blood-2006-11-056291
Downloads
Published
Issue
Section
License
Copyright (c) 2017 National University of Pharmacy
This work is licensed under a Creative Commons Attribution 4.0 International License.
Authors who publish with this journal agree to the following terms:- Authors retain copyright and grant the journal right of first publication with the work simultaneously licensed under a Creative Commons Attribution License that allows others to share the work with an acknowledgement of the work's authorship and initial publication in this journal.
- Authors are able to enter into separate, additional contractual arrangements for the non-exclusive distribution of the journal's published version of the work (e.g., post it to an institutional repository or publish it in a book), with an acknowledgement of its initial publication in this journal.
- Authors are permitted and encouraged to post their work online (e.g., in institutional repositories or on their website) prior to and during the submission process, as it can lead to productive exchanges, as well as earlier and greater citation of published work (See The Effect of Open Access).
