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.