OBJECTIVES: Immune thrombocytopenia (ITP) is an autoimmune disease that reduces platelet count; thus, increases risk of bleeding. ITP is estimated to affect up to 200,000 patients worldwide. Consequently, it imposes over USD 3 billion overall cost on the global economy. Introducing romiplostim, an emerging TPO-RA, has led to positive clinical outcomes in terms of enhancing platelets count. This paper aimed to evaluate the clinical and economic consequences of various treatment pathways of ITP with the addition of romiplostim to the treatment regimens available in Qatar.

METHODS: A cost-utility model was designed to evaluate the long-term effects of romiplostim over lifetime horizon. The model accounted for treatment, monitoring, administration costs, and costs associated with bleeding events. The model compared the costs to quality life-adjusted years (QALY) in three scenarios, (i) romiplostim in early versus romiplostim in chronic ITP, (ii) romiplostim in early ITP versus eltrombopag in chronic ITP, and (iii) romiplostim versus eltrombopag in chronic ITP. A 5-year budget impact analysis was also conducted.

RESULTS: Romiplostim in early ITP was dominant compared to romiplostim in chronic ITP leading to higher QALYs and saving QAR 93,000. Similarly, romiplostim in early ITP was dominant over eltrombopag in chronic ITP, generating more QALYs and saving QAR 23,745. Total costs of romiplostim and eltrombopag in chronic ITP were estimated to be QAR 1,956,297 and QAR 1,883,725, respectively. This yields an incremental cost-effectiveness ratio of QAR 259,185 per QALY with romiplostim, which is less than a willingness-to-pay threshold of 1 GDP per capita. Our model demonstrated that romiplostim led to high drug acquisition cost, yet it reduced overall costs for all other variables.

CONCLUSIONS: Early initiation of romiplostim in ITP is not only clinically effective, but also cost saving compared to late initiation. The budget impact analysis supported the economic outcomes, indicating potential healthcare savings.