Comparative net cost impact of the utilization of romiplostim and intravenous immunoglobulin for the treatment of patients with immune thrombocytopenia in Québec, Canada

Pettigrew, Martine; Garces, Kirsten; Deuson, Robert; Kassis, Jeannine; Laroche, Vinçent

doi:10.3111/13696998.2012.756400

Cited by 11 publications

(7 citation statements)

References 12 publications

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“…In a study from Thailand (a developing country), cost effectiveness of IVIG in childhood ITP has been proven, as compared to standard treatment of thrombocyte transfusions, corticosteroids plus immunosuppressants ( 79 ). On the other hand, health economic studies from Canada and Ireland show for adult chronic ITP patients that romiplostim, a thrombopoietin receptor agonist, seems to compare favorably with standard treatment including IVIG ( 80 , 81 ).…”

Section: Immunomodulatory Therapymentioning

confidence: 99%

Is Dosing of Therapeutic Immunoglobulins Optimal? A Review of a Three-Decade Long Debate in Europe

Kerr

Quinti

Eibl³

et al. 2014

Front. Immunol.

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The consumption of immunoglobulins (Ig) is increasing due to better recognition of antibody deficiencies, an aging population, and new indications. This review aims to examine the various dosing regimens and research developments in the established and in some of the relevant off-label indications in Europe. The background to the current regulatory settings in Europe is provided as a backdrop for the latest developments in primary and secondary immunodeficiencies and in immunomodulatory indications. In these heterogeneous areas, clinical trials encompassing different routes of administration, varying intervals, and infusion rates are paving the way toward more individualized therapy regimens. In primary antibody deficiencies, adjustments in dosing and intervals will depend on the clinical presentation, effective IgG trough levels and IgG metabolism. Ideally, individual pharmacokinetic profiles in conjunction with the clinical phenotype could lead to highly tailored treatment. In practice, incremental dosage increases are necessary to titrate the optimal dose for more severely ill patients. Higher intravenous doses in these patients also have beneficial immunomodulatory effects beyond mere IgG replacement. Better understanding of the pharmacokinetics of Ig therapy is leading to a move away from simplistic “per kg” dosing. Defective antibody production is common in many secondary immunodeficiencies irrespective of whether the causative factor was lymphoid malignancies (established indications), certain autoimmune disorders, immunosuppressive agents, or biologics. This antibody failure, as shown by test immunization, may be amenable to treatment with replacement Ig therapy. In certain immunomodulatory settings [e.g., idiopathic thrombocytopenic purpura (ITP)], selection of patients for Ig therapy may be enhanced by relevant biomarkers in order to exclude non-responders and thus obtain higher response rates. In this review, the developments in dosing of therapeutic immunoglobulins have been limited to high and some medium priority indications such as ITP, Kawasaki’ disease, Guillain–Barré syndrome, chronic inflammatory demyelinating polyradiculoneuropathy, myasthenia gravis, multifocal motor neuropathy, fetal alloimmune thrombocytopenia, fetal hemolytic anemia, and dermatological diseases.

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Section: Immunomodulatory Therapymentioning

confidence: 99%

Is Dosing of Therapeutic Immunoglobulins Optimal? A Review of a Three-Decade Long Debate in Europe

Kerr

Quinti

Eibl³

et al. 2014

Front. Immunol.

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“…Romiplostim has been shown to be cost effective compared with SoC and rituximab in other countries. In Canada, a net cost impact model showed that romiplostim is less expensive to prepare and administer than IVIg and is associated with lower indirect costs from a Canadian healthcare perspective [ 33 ]. Romiplostim was also associated with lower cost per response over 6 months when compared with rituximab from the French national health system perspective [ 34 ] and when compared with SoC in Spain [ 35 ].…”

Section: Discussionmentioning

confidence: 99%

Cost Effectiveness of Romiplostim for the Treatment of Chronic Immune Thrombocytopenia in Ireland

Lee

Thornton

Hirst

et al. 2013

Appl Health Econ Health Policy

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BackgroundRomiplostim, a thrombopoietin receptor agonist (TPOra), is a second-line medical treatment option for adults with chronic immune thrombocytopenia (ITP). Clinical trials have shown that romiplostim increases platelet counts, while reducing the risk of bleeding and, in turn, the need for costly rescue medications.AimsThe objective of this study was to assess the cost effectiveness of romiplostim in the treatment of adult ITP in Ireland, in comparison with eltrombopag and the medical standard of care (SoC).MethodsA lifetime treatment-sequence cost-utility Markov model with embedded decision tree was developed from an Irish healthcare perspective to compare romiplostim with eltrombopag and SoC. The model was driven by platelet response (platelet count ≥50 × 109/L), which determined effectiveness and progression along the treatment pathway, need for rescue therapy (e.g. intravenous immunoglobulin [IVIg] and steroids) and risk of bleeding. Probability of response, mean treatment duration, average time to initial response and utilities were derived from clinical trials and other published evidence. Treatment sequences and healthcare utilization practice were validated by Irish clinical experts. Costs were assessed in € for 2011 and included drug acquisition costs and costs associated with monitoring patients and management of bleeding, as available from published Irish reimbursement lists and other relevant sources. Deterministic and probabilistic sensitivity analyses were conducted.ResultsRomiplostim treatment resulted in an average of 20.2 fewer administrations of rescue medication (IVIg or intravenous steroids) over a patient lifetime than eltrombopag, and 29.3 fewer rescue medication administrations than SoC. Romiplostim was dominant, with cost savings of €13,258 and €22,673 and gains of 0.76 and 1.17 quality-adjusted life-years (QALYs), compared with eltrombopag and SoC, respectively. Romiplostim remained cost effective throughout a variety of potential scenarios, including short-term TPOra treatment duration (1 year). One-way sensitivity analysis showed that the model was most sensitive to variation in the cost of IVIg and use of romiplostim and IVIg. Probabilistic sensitivity analysis showed that romiplostim was likely to be cost effective in over 90 % of cases compared with eltrombopag, and 96 % compared with SoC at a willingness-to-pay threshold of €30,000 per QALY.ConclusionsUse of romiplostim in the ITP treatment pathway, compared with eltrombopag or SoC, is likely to be cost effective in Ireland. Romiplostim improves clinical outcomes by increasing platelet counts, reducing bleeding events and the use of IVIg and steroids, resulting in both cost savings and additional QALYs when compared with current treatment practices.Electronic supplementary materialThe online version of this article (doi:10.1007/s40258-013-0044-y) contains supplementary material, which is available to authorized users.

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“…Romiplostim is administered by subcutaneous injections in a dose based on a patient's body weight and platelet count. Romiplostim, however, is only available in two doses in prefilled vials, 250 µ g and 500 µ g, costing approximately $882.50 and $1765 Cdn, respectively [ 13 ]. The dose is potentially administered indefinitely for patients with chronic ITP.…”

Section: Discussionmentioning

confidence: 99%

Is There a Role for Biweekly Romiplostim in the Management of Chronic Immune Thrombocytopenia (ITP)? A Report of Three Cases

Rooprai

Khamisa

2018

Case Reports in Hematology

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Romiplostim is a peptibody, which stimulates platelet production by a mechanism similar to that of endogenous thrombopoietin. It has an established indication as second-line therapy in patients with chronic immune thrombocytopenia (ITP). The agent is typically administered weekly; however, there are instances where a biweekly (i.e., alternate week) dosing may be feasible in a select group of patients. We conducted a retrospective case review to evaluate the efficacy and safety of biweekly administration of romiplostim in maintaining a platelet count of >30 × 109/L in three patients with chronic ITP. Treatment was started with a weekly injection (1 µg/kg) with a dose escalation to achieve a platelet count >30 × 109/L. Once stable on weekly romiplostim, these patients received biweekly administration. No bleeding complications were noted during biweekly dosing for these patients. The current findings suggest that lengthening the dose interval of romiplostim is feasible in select patients with chronic ITP to maintain stable platelet counts. Additional studies are therefore warranted to further evaluate biweekly dosing for romiplostim to increase convenience and decrease costs for patients with chronic ITP.

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Comparative net cost impact of the utilization of romiplostim and intravenous immunoglobulin for the treatment of patients with immune thrombocytopenia in Québec, Canada

Abstract: Scarce blood products must be used wisely. Romiplostim can allow for improved healthcare resource allocation by reserving IVIg for use in other areas of greater need while also providing cost savings for the overall provincial healthcare budget.

Cited by 11 publications

References 12 publications

Is Dosing of Therapeutic Immunoglobulins Optimal? A Review of a Three-Decade Long Debate in Europe

Is Dosing of Therapeutic Immunoglobulins Optimal? A Review of a Three-Decade Long Debate in Europe

Cost Effectiveness of Romiplostim for the Treatment of Chronic Immune Thrombocytopenia in Ireland

Is There a Role for Biweekly Romiplostim in the Management of Chronic Immune Thrombocytopenia (ITP)? A Report of Three Cases

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