Evaluating the Cost-Effectiveness of Ruxolitinib for the Treatment of Polycythaemia Vera Patients That Are Resistant/Intolerant to Hydroxycarbamide/Hydroxyurea in England and Wales

OBJECTIVES: Polycythaemia vera (PV) is a haematological cancer associated with a substantial and varied symptom burden, as well as a risk of serious complications including thromboembolic and cardiovascular events, disease progression to myelofibrosis (MF), acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS). A considerable percentage of patients develop resistance/intolerance (R/I) to hydroxycarbamide/hydroxyurea (HC/HU). For those patients, 'treatment with ruxolitinib helps control haematocrit, improve quality of life and reduce the incidence of complications. The aim of this analysis is to assess the cost-effectiveness of ruxolitinib compared with current clinical management for patients with PV R/I to HC/HU in England and Wales.

METHODS: A cohort state-transition model was developed composed of four key health states (“progression-free on and off ruxolitinib”, “progressed disease” and “death”). The progressed health state was further split onto transformation into MF or AML/MDS. The impact of thromboembolic events was simplified and included as an event in the form of one-off cost and QALY loss. Efficacy data were taken from the MAJIC-PV trial, supplemented by external data. Utility values were derived from the RESPONSE trials, supplemented by the published literature. The type and frequency of resource use was estimated from clinical experts. The analysis was conducted from the perspective of the NHS and Personal Social Services over a lifetime horizon.

RESULTS: The probabilistic incremental cost per QALY gained was below £20,000, with a 95.6% probability of ruxolitinib being a cost-effective treatment option at a £30,000/QALY gained willingness to pay threshold. Results were robust to changes in structural assumptions and sensitivity analyses.

CONCLUSIONS: In addition to providing a cost-effective option, treatment with ruxolitinib allows patients to be managed away from the hospital, and so may help alleviate NHS capacity issues, reduce the patient burden associated with phlebotomy and improve quality of life for both patients and their families.