NICE has not recommended Novartis’ Jakavi (ruxolitinib) in final draft guidance for the treatment of disease-related splenomegaly or symptoms in adults with myelofibrosis.

An independent appraisal committee concluded the treatment is clinically effective but raised concerns around the manufacturer’s economic model and many of its assumptions.

Sir Andrew Dillon, NICE Chief Executive, said the regulator had to be sure treatments are clinically and cost effective otherwise “money has to be diverted from elsewhere” to fund such drugs in the NHS.

The guidance states that Jakavi is not recommended for the treatment of disease-related cases of an enlarged spleen or its symptoms in adults with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.

Myelofibrosis is a rare type of blood cancer in which the bone marrow produces too many cells too rapidly. This affects the bone marrow making it less able to create cells. Other organs, such as the liver and spleen, then compensate for this by producing additional cells. The spleen, as it begins to create cells, becomes enlarged.

NICE noted that Jakavi was clinically effective in reducing the size of the spleen in these cases and related symptoms. However, it could not be considered a cost-effective option of NHS resources when compared with existing treatments.

The committee found there were “fundamental issues” with the structure of the economic model supplied by Novartis. It concluded that the associated assumptions made increased the uncertainty of the cost-effectiveness ratio (ICER) for the treatment and that rectifying this would actually increase the ICER.

Final guidance is now expected in June 2013.

NICE has issued preliminary guidance not recommending a drug for myelofibrosis, a rare blood cancer, which it says is clinically effective but too costly.

Jakavi (ruxolitinib) from Novartis is provisionally refused for NHS treatment of enlarged spleen caused by myelofibrosis.

Novartis has said it is encouraged by NICE’s acceptance of the drug’s clinical value and will work to reach agreement with the Institute on cost issues.

Myelofibrosis causes scarring of the bone marrow tissue, affecting their ability to produce blood cells. The spleen becomes enlarged to compensate, which has debilitating effects.

The NICE appraisal committee concluded that Jakavi offered a “step change” in treating the condition: it reduced spleen size and symptoms such as fatigue, pain and itching.

However, it did not consider the drug, which costs £43,200 per patient per year, more cost-effective than the next best alternative.

NICE’s value model – recently criticised by the European Commission – sets a threshold price of £30,000 per quality-adjusted life year (QALY) gained. Novartis claims a price per QALY of £74,000, but NICE claims the true figure is twice that.

Professor Carole Longson, Director of NICE’s Health Technology Evaluation Centre, said: “It is disappointing not to be able to recommend this new treatment in our preliminary recommendations, but in order to do this we have to be sure that the treatment is both clinically and cost effective.”

Consultant haematologist Professor Claire Harrison commented: “The lives of patients affected by myelofibrosis are improved with ruxolitinib therapy. In many cases this improvement is dramatic with long-lasting tangible benefits.”

“We are encouraged that the Committee considers ruxolitinib to be an innovative treatment and Novartis is committed to working alongside clinicians and patient groups in this area to address all queries raised by NICE,” said Panos Alexakos, Oncology General Manager, Novartis UK & Ireland.

Final guidance is expected in June 2013.

The CHMP has approved Novartis’ Jakavi (INC424, ruxolitinib) for the rare conditions of adults with myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

The recommendation was based on findings from the COMFORT clinical trials which demonstrated Jakavi to improve overall survival rates and reduce spleen volume compared to placebo.

Hervé Hoppenot, President, Novartis Oncology, said the oral inhibitor “may address unmet patient needs”.

The CHMP’s opinion has now been passed on to the European Commission (EC) to deliver a final decision. The EC has previously granted Jakavi orphan drug designation for myelofibrosis.

Currently, there are only a handful of treatments available to treat the life-threatening blood cancer disease. Myelofibrosis is characterised by multiple severe complications including bone marrow failure, an enlarged spleen and shortened survival.

Myelofibrosis affects around 0.75 out of 100,000 people each year in Europe. Studies have shown that patients with the disease have a decreased life expectancy with a median survival of 5.7 years.

In the COMFORT-I trial, almost half (41.9%) of patients treated with Jakavi achieved at least a 35% reduction in spleen volume at 24 weeks compared to just 0.7% of patients in the placebo group.

In the COMFORT-II trial, the treatment produced a reduction in spleen volume of greater than 35% in 28.5% of patients compared with 0% of patients in the best available therapy – or not therapy at all – group at 48 weeks.

Results also showed how continuous use of the therapy provided a marked and durable improvement in overall quality of life at 48 weeks.

“The Committee’s positive opinion today validates the strong data in support of Jakavi and the very high unmet need in treating myelofibrosis,” said Hervé Hoppenot.

“The recommendation for European Commission approval of Jakavi also marks a significant step forward in bringing the first and only JAK 1 and JAK 2 inhibitor to patients with very limited options.”

A decision is now expected from the EC within the next three months.