OBJECTIVES: In the EU, romiplostim was recently indicated for the treatment of immune thrombocytopenia in patients with disease duration ≤ 12 months (early ITP). In clinical practice, most patients receive romiplostim for chronic ITP. This study assessed the cost-effectiveness of romiplostim in the treatment of early versus chronic ITP in the Czech Republic.

METHODS: A treatment sequence Markov model with lifetime horizon was developed for the cost-effectiveness analysis. In the model, patients switched treatment as they progressed through their disease. Two treatment sequences were compared: 1) romiplostim given in early ITP followed by treatments including rituximab, eltrombopag, and splenectomy; 2) watchful waiting in early ITP followed by romiplostim and the same treatments as in the comparator sequence. Rates of response and treatment-free remission (remission without treatment for 6 months) for romiplostim were estimated from an integrated analysis of clinical trials. Other input parameters were primarily obtained from previous economic evaluations and clinical expert feedback. The model considered a healthcare payer perspective and was populated with Czech cost data (2021 Czech Koruna [CZK]). Main outcomes were life-years (LYs), quality-adjusted LYs (QALYs), costs, and incremental cost-effectiveness ratio.

RESULTS: For romiplostim in early ITP and chronic ITP, the model predicted mean LYs of 17.91 and 17.84 years, QALYs of 14.83 and 14.76 years, and lifetime costs of CZK 15,118,363 and CZK 15,832,694, respectively. The incremental (QA)LYs gain, and costs savings were estimated to be 0.07, and CZK 714,331. Romiplostim in early ITP remained a dominant strategy in extensive sensitivity analyses.

CONCLUSIONS: The cost-effectiveness analysis showed that romiplostim given in early ITP is expected to provide additional LYs and QALYs benefit and saves costs compared with romiplostim given in chronic ITP. Therefore, romiplostim in early ITP is a dominant treatment in the Czech Republic and represents an efficient utilization of the health care budget.