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Cost effectiveness of romiplostim for the treatment of chronic immune thrombocytopenia in Ireland.

Lee D, Thornton P, Hirst A, Kutikova L, Deuson R, Brereton N - Appl Health Econ Health Policy (2013)

Bottom Line: 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 <euro>30,000 per QALY.Use 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.

View Article: PubMed Central - PubMed

Affiliation: BresMed, North Church House, 84 Queen Street, Sheffield, S1 2DW, UK, dlee@bresmed.co.uk.

ABSTRACT

Background: Romiplostim, 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.

Aims: The 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).

Methods: A 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 × 10(9)/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.

Results: Romiplostim 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.

Conclusions: Use 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.

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Related in: MedlinePlus

Markov model with embedded decision tree overview (health states [platelets ≥50 × 109/L, platelets <50 × 109/L, dead] evaluated in 4-week cycles). GI gastrointestinal, W&R watch and rescue, Tn current treatment, Tn + 1 next treatment in sequence
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Related In: Results  -  Collection


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Fig2: Markov model with embedded decision tree overview (health states [platelets ≥50 × 109/L, platelets <50 × 109/L, dead] evaluated in 4-week cycles). GI gastrointestinal, W&R watch and rescue, Tn current treatment, Tn + 1 next treatment in sequence

Mentions: 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 (IVIg and steroids) and risk of bleeding (Fig. 2). Patients started on the first treatment in the pathway and progressed to the next treatment if they did not have an initial response or if they relapsed (platelet count <50 × 109/L) after responding. Each relapse on active treatment, determined by platelet count <50 × 109/L, was followed by a period of ‘watch and rescue’ before initiating the next active treatment. Long-term treatment (keeping patients on active therapy for as long as they had a continued response) was modelled in the base case, and short-term treatment (1 year only) with romiplostim and eltrombopag was tested in a sensitivity analysis.Fig. 2


Cost effectiveness of romiplostim for the treatment of chronic immune thrombocytopenia in Ireland.

Lee D, Thornton P, Hirst A, Kutikova L, Deuson R, Brereton N - Appl Health Econ Health Policy (2013)

Markov model with embedded decision tree overview (health states [platelets ≥50 × 109/L, platelets <50 × 109/L, dead] evaluated in 4-week cycles). GI gastrointestinal, W&R watch and rescue, Tn current treatment, Tn + 1 next treatment in sequence
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

Show All Figures
getmorefigures.php?uid=PMC3824633&req=5

Fig2: Markov model with embedded decision tree overview (health states [platelets ≥50 × 109/L, platelets <50 × 109/L, dead] evaluated in 4-week cycles). GI gastrointestinal, W&R watch and rescue, Tn current treatment, Tn + 1 next treatment in sequence
Mentions: 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 (IVIg and steroids) and risk of bleeding (Fig. 2). Patients started on the first treatment in the pathway and progressed to the next treatment if they did not have an initial response or if they relapsed (platelet count <50 × 109/L) after responding. Each relapse on active treatment, determined by platelet count <50 × 109/L, was followed by a period of ‘watch and rescue’ before initiating the next active treatment. Long-term treatment (keeping patients on active therapy for as long as they had a continued response) was modelled in the base case, and short-term treatment (1 year only) with romiplostim and eltrombopag was tested in a sensitivity analysis.Fig. 2

Bottom Line: 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 <euro>30,000 per QALY.Use 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.

View Article: PubMed Central - PubMed

Affiliation: BresMed, North Church House, 84 Queen Street, Sheffield, S1 2DW, UK, dlee@bresmed.co.uk.

ABSTRACT

Background: Romiplostim, 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.

Aims: The 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).

Methods: A 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 × 10(9)/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.

Results: Romiplostim 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.

Conclusions: Use 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.

Show MeSH
Related in: MedlinePlus