Albutrepenonacog alfa (Idelvion®) for the treatment of Italian patients with hemophilia B: a budget impact model

Lorenzo Pradelli, Sara Villa, Giancarlo Castaman

DOI: https://doi.org/10.7175/fe.v19i1.1328

Abstract

BACKGROUND: Enhanced pharmacokinetic profile of albutrepenonacog alfa allows to prolong the interdose period in prophylaxis, maintaining higher trough level, and to reduce dosage needed for bleeding. This improvement could lead to a better efficiency of the hemophilia B treatment.

OBJECTIVES: To estimate the impact of this new drug on the Italian National Health System (NHS).

METHODS: A model was developed from the NHS perspective to assess the budget impact of treating severe hemophilia B with reimbursed recombinant factor IX over 3 years in Italy. Target population was based on data from the National Registry of Congenital Coagulopathies, which collects data from 54 Hemophilia Treatment Centers. Treatment options were: albutrepenonacog alfa (Idelvion®), eftrenonacog alfa (Alprolix®) and nonacog alfa (BeneFIX®). Annual bleeding rate, dose and infusions needed to treat an episode based on clinical trials data were considered.

RESULTS: Mean costs per patient were calculated for prophylaxis and bleeding treatment by age groups. Applying age-specific costs to the expected new pattern of drugs utilization, the impact on the NHS budget was € 6 million of savings cumulated in 3 years. The model results most sensitive to drug dosages. Lower drug consumption in prophylaxis and reduced bleeding rate than the alternatives reduce expenditures. Main limitations of this analysis were the assumptions that all severe patients receive prophylaxis and the lack of consideration of positive effects of hemorrhagic complications reduction (with consequent lower need of physiotherapy/prosthetic substitution).

CONCLUSIONS: The introduction of Idelvion® as therapeutic option for hemophilia B is expected to decrease pharmaceutical costs and improve patient’s quality of life due to less frequent infusions.

Keywords

Hemophilia B; Recombinant factor IX; Pharmacokinetics; Budget impact

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References

  • Treatment Guidelines Working Group, on behalf of the World Federation of Hemophilia (WFH). Guidelines for the Management of hemophilia - 2nd edition. World Federation of Hemophilia: Montréal, 2013. Available at http://www1.wfh.org/publications/files/pdf-1472.pdf (last accessed June 2017)
  • Abbonizio F, Giampaolo A, Arcieri R, et al.;Associazione Italiana Centri Emofilia (AICE). Registro Nazionale Coagulopatie Congenite. Rapporto 2014. Roma: Istituto Superiore di Sanità; 2016 (Rapporti ISTISAN 16/20)
  • Kodra Y, Cavazza M, Schieppati A, et al. The social burden and quality of life of patients with haemophilia in Italy. Blood Transfus 2014; 12 Suppl 3: s567-75; https://doi.org/10.2450/2014.0042-14s
  • Rocino A, Coppola A, Franchini M, et al.; Italian Association of Haemophilia Centres (AICE) Working Party. Principles of treatment and update of recommendations for the management of haemophilia and congenital bleeding disorders in Italy. Blood Transfus 2014; 12: 575-98. https://doi.org/10.2450/2014.0223-14
  • Valentino LA, Rusen L, Elezovic I, et al. Multicentre, randomized, open-label study of on-demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects. Haemophilia 2014; 20: 398-406; https://doi.org/10.1111/hae.12344
  • Kavakli K, Smith L, Kuliczkowski K, et al. Once-weekly prophylactic treatment vs. on-demand treatment with nonacog alfa in patients with moderately severe to severe haemophilia B. Haemophilia 2016; 22: 381-8; https://doi.org/10.1111/hae.12878
  • Powell JS, Pasi KJ, Ragni MV, et al.; B-LONG Investigators. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med 2013; 369: 2313-23; https://doi.org/10.1056/NEJMoa1305074
  • Fischer K, Kulkarni R, Nolan B, et al. Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study. Lancet Haematol 2017; 4: e75-e82; https://doi.org/10.1016/S2352-3026(16)30193-4
  • Santagostino E, Negrier C, Klamroth R, et al. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood 2012; 120: 2405-11; https://doi.org/10.1182/blood-2012-05-429688
  • Martinowitz U, Lissitchkov T, Lubetsky A, et al. Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients. Haemophilia 2015; 21 784-90; https://doi.org/10.1111/hae.12721
  • Santagostino E, Martinowitz U, Lissitchkov T, et al.; PROLONG-9FP Investigators Study Group. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood 2016; 127: 1761-9; https://doi.org/10.1182/blood-2015-09-669234
  • Kenet G, Chambost H, Male C,; PROLONG-9FP Investigator Study Group. Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children. Results of a phase 3 trial. Thromb Haemost 2016; 116: 659-68; https://doi.org/10.1160/TH16-03-0179
  • EMA. Committee for Medicinal Products for Human Use (CHMP). Assessment report – Idelvion®. EMA/CHMP/213825/2016. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/003955/WC500207382.pdf (last accessed June 2017)
  • Idelvion® – Summary of Product Characteristics. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/003955/WC500207380.pdf (last accessed June 2017)
  • EMA. Committee for Orphan Medicinal Products. Public summary of opinion on orphan designation. Recombinant fusion protein linking human coagulation factor IX with human albumin for the treatment of hemophilia B. EMA/COMP/816162/2009. Available at http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2010/03/WC500075161.pdf (last accessed June 2017)
  • EMA. Procedure Management and Committees Support Division. Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation EMA/COMP/150969/2016. Available at http://www.ema.europa.eu/docs/en_GB/document_library/Committee_meeting_report/2016/04/WC500204294.pdf (last accessed June 2017)
  • EMA. Idelvion® - albutrepenonacog alfa. Authorization details. Available at http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/003955/human_med_001974.jsp&mid=WC0b01ac058001d124 (last accessed June 2017)
  • Zhang Y, Roberts J, Bensen-Kennedy D, et al. Population pharmacokinetics of a new long-acting recombinant coagulation factor IX albumin fusion protein for patients with severe hemophilia B. J Thromb Haemost 2016; 14: 2132-40; https://doi.org/10.1111/jth.13444
  • Santagostino E, Voigt C, Wolko D, et al. Interim results of a Phase IIIb safety and efficacy extension study of a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in patients with hemophilia B. WFH 2016 World Congress. Orlando, 24-28 July 2016. Available at https://www.postersessiononline.eu/173580348_eu/congresos/WFH2016/aula/-PP-T_113_WFH2016.pdf (last accessed June 2017)
  • ISTAT. Growth rate for Italian male population. Available at www.demo.istat.it (last accessed June 2017)
  • Associazione Italiana Centri Emofilia (AICE). Available at http://www.aiceonline.org/ (last accessed June 2017)
  • Informatore Farmaceutico 2017.Available at www.codifa.it (last accessed March 2017)
  • Alprolix® – Prescribing information. Available at www.alprolix.com/pdfs/PrescribingInformation.pdf (last accessed June 2017)
  • Iorio A, Krishnan S, Myrén KJ, et al. Continuous prophylaxis with recombinant factor IX Fc fusion protein and conventional recombinant factor IX products: comparisons of efficacy and weekly factor consumption. J Med Econ 2017; 20: 337-344; https://doi.org/10.1080/13696998.2016.1265973
  • European Medicine Agency. Committee for Medicinal Products for Human Use (CHMP). Assessment report – Alprolix®. EMA/CHMP/196953/2016. Available at http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/004142/WC500207016.pdf (last accessed June 2017)
  • Powell J, Shapiro A, Ragni M, et al. Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates. Br J Haematol 2015; 168: 113-23; https://doi.org/10.1111/bjh.13109
  • European Medicine Agency. Committee for Medicinal Products for Human Use (CHMP). Assessment report – BeneFIX®. EMA/37098/2013 . Available at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Assessment_Report_-_Variation/human/000139/WC500141970.pdf (last accessed June 2017)
  • Benefix® – Prescribing information. Available at labeling.pfizer.com/showlabeling.aspx?id=492 (last accessed June 2017)
  • National Hemophilia Foundation. Hemophilia B. 2015. Available at http://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-B (last accessed June 2017)

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