ruxolitinib (Jakavi)

following a full submission considered under the orphan process.

ruxolitinib (Jakavi®) is accepted for use within NHS Scotland.

Indication under review: the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.

In patients with myelofibrosis, a significantly greater proportion of patients achieved a spleen response (reduction in spleen volume of at least 35% from baseline) at 48 weeks when treated with ruxolitinib compared with best available therapy. Ruxolitinib was also associated with a greater proportion of patients reporting a clinically significant reduction in myelofibrosis-related symptoms when compared with placebo.

This advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost effectiveness of ruxolitinib. It is contingent upon the continuing availability of the Patient Access Scheme in NHS Scotland or a list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.