Skip to main content
Search
Search
Advanced Search
Skip main navigation
Free access
Research Article
31 May 2018

A decision framework for treating chronic immune thrombocytopenia with thrombopoietin receptor agonists

Authors: Mike Dolph, Anuja Roy, Menaka Bhor, Jaclyn Hearnden, Christina S Kwon, Anna Forsythe [email protected], Gabriel Tremblay, and Andrew BriggsAuthor Info & Affiliations
Volume 7, Number 8
https://doi.org/10.2217/cer-2018-0034
PDFFormats

Abstract

Aim: Eltrombopag and romiplostim are comparable second-line therapies in chronic immune thrombocytopenia. Treatment decisions are made in different contexts. A framework was created to outline decision pathways for physicians and payers. Materials & methods: The costs of drugs, administration, routine care, bleeding, other adverse events and mortality were included in the year-long calculation of total costs from a US private payer perspective. Treatment parameters and outcome data were obtained from relevant clinical trials. Results: The total cost per year, per patient of eltrombopag was US$51,000 versus US$76,000 for romiplostim. Drug costs and costs associated with bleeding-related events were the main drivers of cost difference. Conclusion: This framework facilitates decision-making in the management of chronic immune thrombocytopenia with eltrombopag and romiplostim.

Supplementary Material

File (supplementaryinformation.docx)

References

Papers of special note have been highlighted as: • of interest; •• of considerable interest
1.
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 113(11), 2386–2393 (2009).
PubMed
Google Scholar
2.
Segal JB, Powe NR. Prevalence of immune thrombocytopenia: analyses of administrative data. J. Thromb. Haemost. 4(11), 2377–2383 (2006).
PubMed
Google Scholar
3.
Landgren O, Gridley G, Fears TR, Caporaso N. Immune thrombocytopenic purpura does not exhibit a disparity in prevalence between African American and white veterans. Blood 108(3), 1111–1112 (2006).
PubMed
Google Scholar
5.
Provan D, Newland AC. Current management of primary immune thrombocytopenia. Adv. Ther. 32(10), 875–887 (2015).
PubMed
Google Scholar
6.
Stevens W, Koene H, Zwaginga JJ, Vreugdenhil G. Chronic idiopathic thrombocytopenic purpura: present strategy, guidelines and new insights. Neth. J. Med. 64(10), 356–363 (2006).
PubMed
Google Scholar
7.
Mathias SD, Gao SK, Miller KL et al. Impact of chronic immune thrombocytopenic purpura (ITP) on health-related quality of life: a conceptual model starting with the patient perspective. Health Qual. Life Outcomes 6, 13 (2008).
PubMed
Google Scholar
8.
Zhou Z, Yang L, Chen Z et al. Health-related quality of life measured by the Short Form 36 in immune thrombocytopenic purpura: a cross-sectional survey in China. Eur. J. Haematol. 78(6), 518–523 (2007).
PubMed
Google Scholar
9.
Michel M. Immune thrombocytopenic purpura: epidemiology and implications for patients. Eur. J. Haematol. Suppl. (71), 3–7 (2009)
PubMed
Google Scholar
10.
Lin J, Zhang X, Li X et al. Cost of bleeding-related episodes in adult patients with primary immune thrombocytopenia: a population-based retrospective cohort study of administrative claims data for commercial payers in the United States. Clin. Ther. 39(3), 603.e601–609.e601 (2017).
Google Scholar
•• Reports on the high costs of hospitalization due to bleeding-related events in immune thrombocytopenia. Bleeding costs in this analysis were based on this study by Lin et al.
11.
Saleh MN, Fisher M, Grotzinger KM. Analysis of the impact and burden of illness of adult chronic ITP in the US. Curr. Med. Res. Opin. 25(12), 2961–2969 (2009).
PubMed
Google Scholar
• Estimates by Saleh et al. and Danese et al. informed routine care and mortality costs in this analysis.
12.
Danese MD, Lindquist K, Gleeson M, Deuson R, Mikhael J. Cost and mortality associated with hospitalizations in patients with immune thrombocytopenic purpura. Am. J. Hematol. 84(10), 631–635 (2009).
PubMed
Google Scholar
• Estimates by Saleh et al. and Danese et al. informed routine care and mortality costs in this analysis.
13.
Provan D, Newland AC. Guidelines for immune thrombocytopenia. N. Engl. J. Med. 364(6), 580–581 (2011).
PubMed
Google Scholar
14.
Li X, Sharma A, Zhang X et al. Cost per treatment success of thrombopoietin receptor agonists vs “watch and rescue” strategy for treating adult non-splenectomized patients with chronic immune thrombocytopenia: a US payer perspective. Blood 126(23), 4461–4461 (2015).
Google Scholar
15.
Portielje JE, Westendorp RG, Kluin-Nelemans HC, Brand A. Morbidity and mortality in adults with idiopathic thrombocytopenic purpura. Blood 97(9), 2549–2554 (2001).
PubMed
Google Scholar
16.
George JN. Management of patients with refractory immune thrombocytopenic purpura. J. Thromb. Haemost. 4(8), 1664–1672 (2006).
PubMed
Google Scholar
17.
Moulis G, Sailler L, Sommet A, Lapeyre-Mestre M, Derumeaux H, Adoue D. Rituximab versus splenectomy in persistent or chronic adult primary immune thrombocytopenia: an adjusted comparison of mortality and morbidity. Am. J. Hematol. 89(1), 41–46 (2014).
PubMed
Google Scholar
18.
Cheng G, Saleh MN, Marcher C et al. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, Phase 3 study. Lancet 377(9763), 393–402 (2011).
PubMed
Google Scholar
•• Cheng et al. and Kuter et al. provided data on the efficacy of eltrombopag and romiplostim, respectively.
19.
Kuter DJ, Bussel JB, Lyons RM et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet 371(9610), 395–403 (2008).
PubMed
Google Scholar
•• Cheng et al. and Kuter et al. provided data on the efficacy of eltrombopag and romiplostim, respectively.
20.
Consumer Price Index. United States Department of Labor: Bureau of Labor Statistics, 2017. www.bls.gov/cpi/data.htm
Google Scholar
21.
Kuter DJ, Rummel M, Boccia R et al. Romiplostim or standard of care in patients with immune thrombocytopenia. N. Engl. J. Med. 363(20), 1889–1899 (2010).
PubMed
Google Scholar
22.
Tremblay G DM, Bhor M, Elliott B. Cost consequence model comparing eltrombopag and romiplostim for adult patients with previously-treated chronic immune thrombocytopenia. Presented at: ISPOR 22nd Annual International Meeting. Boston, MA, USA, 20 May 2017.
Google Scholar
23.
Electronic Medical Records. Federated network of large hospital institutions across the US. 2017.
Google Scholar
24.
Cooper K, Matcham J, Helme K, Akehurst R. Update on romiplostim and eltrombopag indirect comparison. Int. J. Technol. Assess. Health Care 30(1), 129–130 (2014).
PubMed
Google Scholar
25.
Eltrombopag for treating chronic immune (idiopathic) thrombocytopenic purpura. In: Technology Appraisal Guidance [TA293]. National Institute for Health and Care Excellence (NICE), (2013). www.nice.org.uk/guidance/ta293
Google Scholar
26.
First Databank Drug Pricing Database In: Analysource: Premier Drug Pricing Services. Analysource, Syracuse, NY, USA (2017). www.analysource.com/
Google Scholar
27.
Centers for Medicare and Medicaid Services Fee Schedule, 2017. www.cms.gov/Medicare/Medicare-Fee-for-Service-Payment/PhysicianFeeSched/
Google Scholar
28.
Bach PB, Conti RM, Muller RJ, Schnorr GC, Saltz LB. Overspending driven by oversized single dose vials of cancer drugs. BMJ 352, i788 (2016).
PubMed
Google Scholar
29.
Forsythe A, Bhor M, Roy A, Portella MSO, Kwon C, Tremblay G. Economic burden of immune thrombocytopenia (ITP) in patients receiving eltrombopag (EPAG) and romiplostim (ROMI): real world evidence from 26 US institutions. Presented at: ISPOR 20th Annual European Congress. Glasgow, Scotland, 8 November 2017.
Google Scholar
30.
Forsythe A, Roy A, Bhor M, Portella MSO. Bleeding-related episodes (BRE) in patients with immune thrombocytopenia (ITP) receiving eltrombopag (EPAG) or romiplostim (ROMI): real world evidence from 26 US institutions. Presented at: ISPOR 20th Annual European Congress. Glasgow, Scotland, 8 November 2017.
Google Scholar
31.
Kwon C, Forsythe A, Roy A, Bhor M, Portella MSO, Tremblay G. Health care resource use (HCRU) due to bleeding related episodes (BRE) in patients with immune thrombocytopenic purpura (ITP) receiving eltrombopag (EPAG), romiplostim (ROMI), or rituximab (RITUX): real world evidence (RWE) from 27 US institutions. Presented at: ISPOR 20th Annual European Congress. Glasgow, Scotland, 8 November 2017.
Google Scholar
32.
Forsythe A, Roy A, Bhor M et al. Benefit–risk comparison in patients with immune thrombocytopenia (ITP) receiving eltrombopag or romiplostim: real world evidence (RWE) from 26 hospital institutions. Presented at: ASH 59th Annual Meeting and Exposition. Atlanta, GA, USA, 6 December 2017.
Google Scholar
33.
HCUPnet Healthcare Cost and Utilization Project. AHRQ Agency for Healthcare Research (2017). https://hcupnet.ahrq.gov/#setup
Google Scholar
34.
Josephson CD, Granger S, Assmann SF et al. Bleeding risks are higher in children versus adults given prophylactic platelet transfusions for treatment-induced hypoproliferative thrombocytopenia. Blood 120(4), 748–760 (2012).
PubMed
Google Scholar
35.
WHO Handbook for Reporting the Results of Cancer Treatment. WHO, Geneva, Switzerland (1979). http://apps.who.int/iris/handle/10665/37200
Google Scholar
36.
CHMP ASSESSMENT REPORT FOR Nplate, International Nonproprietary Name: Romiplostim. In: Procedure No. EMEA/H/C/942. European Medicines Agency Evaluation of Medicines for Human Use, London, UK (2015) www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/000942/WC500039475.pdf
Google Scholar
37.
Allen R, Bryden P, Grotzinger KM, Stapelkamp C, Woods B. Cost–effectiveness of eltrombopag versus romiplostim for the treatment of chronic immune thrombocytopenia in England and Wales. Value Health 19(5), 614–622 (2016).
PubMed
Google Scholar
|