NICE has confirmed it is not recommending the use of a drug to treat disease-related splenomegaly - enlarged spleen - on the NHS.
It has published final draft guidance detailing its decision on the Novartis drug Jakavi (ruxolitinib) for the treatment of disease-related splenomegaly or its symptoms in adults with primary myelofibrosis, a rare blood cancer.
The decision also related to those with post polycythaemia vera myelofibrosis and post essential thrombocythaemia myelofibrosisi.
In patients with myelofibrosis, the bone marrow produces too many cells too quickly, causing the marrow to become scarred and less able to produce blood cells, which prompts other organs such as the liver and spleen to compensate by producing them.
The spleen then becomes enlarged as it starts to produce blood cells.
Ruxolitinib was found by the independent committee to be clinically effective in reducing spleen size and symptoms such as itch and fatigue.
It concluded, however, that it was not a more cost-effective option than the best therapy currently available.
The committee found plausibility in the suggestion that the drug could offer survival benefits but noted uncertainties in the evidence, which failed to establish why this may be the case.
It also noted fundamental issues surrounding the manufacturer’s economic model for the drug, which in turn impacted on its cost-effectiveness.
NICE chief executive Sir Andrew Dillon expressed disappointment at not being able to recommend the drug and acknowledged the symptoms of itching and fatigue associated with myelofibrosis and splenomegaly “can be extremely debilitating”.
However, he stressed that to recommend it “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”.