Pharmacoeconomic Analysis of the Use of Thrombopoietin Receptors Agonists in Idiopathic Thrombocytopenic Purpura Therapy

VA Shuvaev1, SV Voloshin1, AK Hadzhidis2, AV Chechetkin1

1Russian Research Institute of Hematology and Transfusiology, 16 2-ya Sovetskaya str., Saint Petersburg, Russian Federation, 191024

2Saint Petersburg State Pediatric Medical University, 2 Litovskaya str., Saint Petersburg, Russian Federation, 194100

For correspondence: Vasilii Anatol’evich Shuvaev, PhD, 16 2-ya Sovetskaya str., Saint Petersburg, Russian Federation, 191024; e-mail: shuvaev77@mail.ru

For citation: Shuvaev VA, Voloshin SV, Khadzhidis AK, Chechetkin AV. Pharmacoeconomic Analysis of the Use of Thrombopoietin Receptors Agonists in Idiopathic Thrombocytopenic Purpura Therapy. Clinical oncohematology. 2017;10(4):435–42 (In Russ).

DOI: 10.21320/2500-2139-2017-10-4-435-442


ABSTRACT

Background. New medications, thrombopoietin mimetics which were recently introduced into clinical practice allowed to achieve clinical response in patients with chronic glucocorticoid-resistant idiopathic thrombocytopenic purpura (ITP). However, the high cost and the need for long-term administration necessitate a pharmacoeconomic analysis of the use of thrombopoietin receptors agonists in the treatment of ITP.

Aim. To assess the cost-effectiveness of the use of thrombopoietin mimetics (romiplostim and eltrombopag) and immunosuppressive therapy in the treatment of chronic glucocorticoid-resistant ITP.

Materials & Methods. The Markov modelling of diagnosis and treatment of ITP was conducted in accordance with the National guidelines for diagnosis and treatment of primary ITP. The cost-benefit analysis of the use of thrombopoietin receptors agonists (romiplostim and eltrombopag) and immunosuppressive therapy was performed. The time period (horizon) of the study was 5 years.

Results. The therapy with thrombopoietin mimetics had higher costs but was shown to be more effective compared to immunosuppressive therapy. The cost-effectiveness for achieving 1 QALY in the treatment was 1.33 million rubles with eltrombopag, 4.2 million rubles with romiplostim, and 0.17 million rubles with immunosuppressive therapy. The lowest additional costs compared to immunosuppressive therapy had eltrombopag treatment, whereas romiplostim treatment doubled the additional costs. The threshold values of the ratio of thrombopoietin receptors agonists costs were determined for the cost-benefit analysis. The use of romiplostim is cost-effective at a price for 1 vial of 15–18 % less than for 1 package of eltrombopag. The total cumulative burden of treatment of chronic ITP for 5 years may be 7.18 billion rubles with the use of eltrombopag, 23.23 billion rubles with romiplostim, and 0.91 billion rubles with immunosuppressive therapy only. The results confirm the need for budgeting the diagnosis and treatment of ITP not as a part of general approach, but to consider ITP as an orphan disease.

Conclusion. The developed pharmacoeconomic model can be used as an assessment tool of the costs of new diagnostic approaches and treatment strategies and optimizing budget expenditures.

Keywords: idiopathic thrombocytopenic purpura, romiplostim, eltrombopag, cost-effectiveness.

Received: May 15, 2017

Accepted: August 7, 2017

Read in PDF


REFERENCES

  1. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115(2):168–86. doi: 10.1182/blood-2009-06-225565.
  2. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an International Working Group. Blood. 2009;113(11):2386–93. doi: 10.1182/blood-2008-07-162503.
  3. Меликян А.Л., Пустовая Е.И., Цветаева Н.В. и др. Национальные клинические рекомендации по диагностике и лечению первичной иммунной тромбоцитопении (идиопатической тромбоцитопенической пурпуры) у взрослых. Гематология и трансфузиология. 2015;60(1):44–56.Melikyan AL, Pustovaya EI, Tsvetaeva NV, et al. National clinical guidelines for еру diagnosis and treatment of primary immune thrombocytopenia (idiopathic thrombocytopenic purpura) in adult patients. Gematologiya i transfuziologiya. 2015;60(1):44–56. (In Russ)]
  4. Лисуков И.А., Масчан А.А., Шамардина А.В. и др. Иммунная тромбоцитопения: клинические проявления и ответ на терапию. Промежуточный анализ данных Российского регистра пациентов с первичной иммунной тромбоцитопенией и обзор литературы. Онкогематология. 2013;8(2);61–9. doi: 10.17650/1818-8346-2013-8-2-61-69.[Lisukov IA, Maschan AA, Shamardina AV, et al. Immune thrombocytopenia: clinical manifestation and therapy response. The interim analysis of Russian register of patients with primary immune thrombocytopenia and literature review. Onkogematologiya. 2013;8(2);61–9. doi: 10.17650/1818-8346-2013-8-2-61-69. (In Russ)]
  5. Rodeghiero F, Michel M, Gernsheimer T, et al. Standardization of bleeding assessment in immune thrombocytopenia: report from the International Working Group. Blood. 2013;121(14):2596–606. doi: 10.1182/blood-2012-07-442392.
  6. Масчан А.А., Румянцев А.Г. Стимуляция продукции тромбоцитов: новый подход к лечению хронической иммунной тромбоцитопенической пурпуры. Онкогематология. 2009:1:51–6.[Maschan AA, Rumyantsev AG. Stimulation of platelet production: The new treatment approach to chronic immune thrombocytopenic purpura. Onkogematologiya. 2009:1:51–6. (In Russ)]
  7. Kuter DJ, Bussel JB, Newland A, et al. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013;161(3):411–23. doi: 10.1111/bjh.12260.
  8. Garnock-Jones KP, Keam SJ. Eltrombopag. Drugs. 2009;69(5):567–76. doi: 10.2165/00003495-200969050-00005.
  9. Bussel JB, Cheng G, Saleh MN, et al. Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura. N Engl J Med. 2007;357(22):2237–47. doi: 10.1056/NEJMoa073275.
  10. Меликян А.Л., Пустовая Е.И., Цветаева Н.В. Национальные клинические рекомендации по диагностике и лечению первичной иммунной тромбоцитопении (идиопатической тромбоцитопенической пурпуры) у взрослых [электронный документ]. Доступно по: http://npngo.ru/wp-content/uploads/2016/09/nacionalnye-klinicheskie-rekomendacii-po-diagnostike-i-lecheniyu-idiopaticheskoj-trombocitopenicheskoj-purpury-pervichnoj-immunnoj-trombocitopenii-u-vzroslyx.pdf. Ссылка активна на 31.07.2017.[Melikyan AL, Pustovaya EI, Tsvetaeva NV, et al. National clinical guidelines for diagnosis and treatment of primary immune thrombocytopenia (idiopathic thrombocytopenic puprura) in adult patients [Internet]. Available from: http://npngo.ru/wp-content/uploads/2016/09/nacionalnye-klinicheskie-rekomendacii-po-diagnostike-i-lecheniyu-idiopaticheskoj-trombocitopenicheskoj-purpury-pervichnoj-immunnoj-trombocitopenii-u-vzroslyx.pdf. (accessed 31.07.2017) (In Russ)]
  11. Воробьев А.П. Клинико-экономический анализ. М.: Ньюдиамед, 2008. 778 с.[Vorob’ev AP. Kliniko-ekonomicheskii analiz. (Clinico-economic analysis.) Moscow: N’yudiamed Publ.; 2008. 778 p. (In Russ)]
  12. Федеральная служба государственной статистики [электронный документ]. Доступно по: http://www.gks.ru/wps/wcm/connect/rosstat_main/rosstat/ru/statistics/accounts/#. Ссылка активна на 31.07.2017.[Federal Service of State statistics [Internet]. Available from: http://www.gks.ru/wps/wcm/connect/rosstat_main/rosstat/ru/statistics/accounts/#. (accessed 31.07.2017) (In Russ)]
  13. Ковалева Л.Г., Сафонова Т.И., Колосова Е.Н. и др. Клинико-статистические данные и оценка различных методов терапии идиопатической тромбоцитопенической пурпуры. Терапевтический архив. 2011;4:60–5.[Kovaleva LG, Safonova TI, Kolosova EN, et al. Clinical statistics and effectiveness of different treatments of idiopathic thrombocytopenic purpura. Terapevticheskii arkhiv. 2011;4:60–5. (In Russ)]
  14. Szende A, Brazier J, Schaefer C, et al. Measurement of utility values in the UK for health states related to immune thrombocytopenic purpura. Curr Med Res Opin. 2010;26(8):1893–903. doi: 10.1185/03007995.2010.494126.
  15. Sanz MA, Aledort L, Mathias SD, et al. Analysis of EQ-5D scores from two phase 3 clinical trials of romiplostim in the treatment of immune thrombocytopenia (ITP). Value in Health. 2011;14(1):90–6. doi: 10.1016/j.jval.2010.10.017.
  16. National Institute for Health and Care Excellence. Romiplostim for the treatment of chronic immune (idiopathic) thrombocytopenic purpura. NICE technology appraisal guidance [TA221] 27.04.2011 [cited 2014 May 1]. Available from: https://www.nice.org.uk/guidance/ta221. Accessed 31.07.2017.
  17. Boyers D, Jia X, Jenkinson D, et al. Eltrombopag for the Treatment of Chronic Immune or Idiopathic Thrombocytopenic Purpura. PharmacoEconomics. 2012;30(6):483–95. doi: 10.2165/11591550-000000000-00000.
  18. Официальный сайт единой информационной системы в сфере закупок [электронный документ]. Доступно по: . Ссылка активна на 31.07.2017.[Official website of the United information system of state purchases [Internet]. Available from: . (accessed 31.07.2017) (In Russ)]
  19. Жулев Ю. У закона нет сирот. Российская газета: Фармацевтика. 2012;5717(44) [электронный документ]. Доступно по: https://rg.ru/2012/03/01/zakon.html. Ссылка активна на 31.07.2017.[Zhulev Y. The law has no orphans. Rossiiskaya gazeta: Farmatsevtika. 2012;5717(44) [Internet]. Available from: https://rg.ru/2012/03/01/zakon.html. (accessed 31.07.2017) (In Russ)]
  20. National Institute for Health and Care Excellence. Eltrombopag for treating chronic immune (idiopathic) thrombocytopenic purpura. NICE technology appraisal guidance [TA293] 24.07.2013 [cited 2016 Sep 3]. Available from: https://www.nice.org.uk/guidance/ta293. Accessed 31.07.2017.