Ruxolitinib for treating disease-related splenomegaly or symptoms in adults with myelofibrosis

Ruxolitinib (Jakavi, Novartis) is a protein kinase inhibitor that targets Janus-associated kinase (JAK) signalling. Ruxolitinib has a UK marketing authorisation for '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'. It is administered orally. The recommended starting dose is 15 mg twice daily for patients with a platelet count between 100,000/mm³ and 200,000/mm³, and 20 mg twice daily for patients with a platelet count of more than 200,000/mm³. The maximum recommended starting dose for patients with platelet counts between 50,000/mm³ and 100,000/mm³ is 5 mg twice daily.

The summary of product characteristics lists the following adverse reactions for ruxolitinib: anaemia, thrombocytopenia, neutropenia, bleeding and weight gain. For full details of adverse reactions and contraindications, see the summary of product characteristics.

The cost of ruxolitinib is £3,360 for a 56‑tablet pack of 10 mg, 15 mg or 20 mg tablets, or £1,680 for a 56‑tablet pack of 5 mg tablets (BNF, December 2015). This amounts to an annual cost of about £43,680 per patient (assuming a 15 mg or 20 mg dose, taken twice daily, for 52 weeks). The company has agreed a patient access scheme with the Department of Health. This scheme provides a simple discount to the list price of ruxolitinib with the discount applied at the point of purchase or invoice. The level of the discount is commercial in confidence. The Department of Health considered that this patient access scheme does not constitute an excessive administrative burden on the NHS.