Posted 26 June 2013
By Louise Zornoza, RegLink
In final draft guidance released today, the UK's National Institute for Health and Care Excellence (NICE) has recommended against the use of ruxolitinib (Jakavi, Novartis) for the treatment of disease-related splenomegaly (enlarged spleen) or its symptoms in adults with primary myelofibrosis, post polycythaemia vera myelofibrosis, or post essential thrombocythaemia myelofibrosisi.
Myelofibrosis is a rare blood cancer in which the bone marrow produces too many cells too rapidly and becomes scarred, making it less able to make blood cells. To compensate for this, other organs in the body, including the liver and the spleen, begin to produce them. As the spleen begins to produce blood cells, it becomes enlarged.
The group of experts which developed the recommendations for NICE concluded that although ruxolitinib was clinically effective in reducing spleen size and symptoms such as itch and fatigue, it could not be considered a cost-effective use of NHS resources compared with best available therapy for treating disease-related splenomegaly or symptoms in adults with myelofibrosis. It concluded that although it was plausible that ruxolitinib could offer an overall survival benefit, the reason for this remained unclear because of uncertainties in the evidence.
Commenting on the draft guidance, NICE Chief Executive Sir Andrew Dillon said: "Myelofibrosis and splenomegaly can be extremely debilitating, with symptoms such as severe itching and fatigue. It is disappointing not to be able to recommend this new treatment in our final draft guidance, but in order to do this we have to be sure that the treatment is both clinically and cost effective, because money has to be diverted from elsewhere in the health service to pay for it."
The Committee noted the Evidence Review Group's concerns around the limitations of the manufacturer's economic model and many of the modeling assumptions which increased the uncertainty of the incremental cost-effectiveness ratio.
NICE Final Draft Guidance on Jakavi
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