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 FDA has approved Abbott Molecular’s genetic diagnostic, Vysis EGR1 FISH Probe Kit, to detect acute myeloid leukaemia (AML).

The new in vitro test is the third of Abbott’s FISH assays to be cleared for oncology applications in the US in the past two months.

Stafford O'Kelly, Head of Abbott's Molecular Diagnostics Business said that the medical technology “can identify which AML patients have the chromosomal abnormality upon diagnosis and provides physicians with another clinically validated tool to assess a patient's overall prognosis”.

The Kit’s technology detects chromosomal deletion in the bone marrow, and can be used in addition to cytogenetics, other biomarkers, morphology and other clinical information, at the time of AML diagnosis to determine prognosis.

Each year, more than 12,000 people are diagnosed with AML, which is a rapidly progressive disease. Current standard procedure involves aggressive chemotherapy drugs, while some patients may require a stem-cell transplant to replace unhealthy bone marrow with leukaemia-free stem cells.  

Part of Abbott, Abbott Molecular is a leader in molecular diagnostics, analysing DNA and RNA at the molecular level. Abbott Molecular's tests aim for earlier detection or diagnosis, to provide information of appropriate treatment, and improve monitoring of disease progression.

Abbott is a global healthcare company, which discovers, develops, manufactures and markets pharmaceuticals and medical products, including nutritionals, devices and diagnostics. The company employs nearly 90,000 people and commercialises its products in more than 130 countries worldwide.

The FDA has granted Orphan Drug Designation to Revimmune for the prevention of graft-versus-host disease (GVHD) following bone marrow transplant.

The treatment, manufactured by Accentia Biopharmaceuticals and its subsidiary Biovest, is believed to destroy mature lymphocytes throughout the body whilst sparing healthy stem cells in the bone marrow.

US Congressman C.W. Young, who initiated the development of the National Marrow Donor Program in the US, says the “new therapy may improve the success rate of many bone marrow transplants”.

Revimmune is based on an extremely high dose of Cytoxan, Baxter Healthcare’s branded cyclophosphamide. The company has agreed for the worldwide commercialisation of Cytoxan, to treat a range of autoimmune diseases including multiple sclerosis as well as the prevention of GVHD following bone marrow transplant.

Mr Young added: “Bone marrow transplantation offers the gift of life to thousands of men, women and children with otherwise fatal blood disorders, and I support everything we can do to give these patients the best chance at success.”

GVHD is a life-threatening autoimmune complication that can occur following a stem cell or bone marrow transplant, where the new cells cause an attack on the patient’s immune system.

Florida-based Accentia Biopharmaceuticals manufactures advanced therapy options involving autoimmune diseases.

NICE has recommended a discounted version of Tasigna (nilotinib, pictured) for the treatment of chronic and accelerated phases of chronic myeloid leukaemia (CML) that is resistant or intolerant to standard dose Glivec, in new draft guidance.

But the Institute has failed to recommend Sprycel (dasatinib) despite saying it is equally as effective as Tasigna, and high-dose Glivec for the same condition.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE, says that both treatments are “expensive” but Novartis’ discount “enabled the independent Committee to approve” its use on the NHS.

Both Tasigna and Sprycel cost over £30,000 per patient, per year. Novartis recently increased the price to high-dose Glivec to £40,000 per patient, per year, although it requested to keep discounted price of Tasigna confidential.

CML is a very rare condition that affects approximately 560 people each year in the UK. If the standard treatment option Glivec does not work, current recommended alternatives are are interferon-alfa, hydroxycarbamide, or a bone marrow transplant.

“We are very pleased to be able to recommend nilotinib as a treatment option for the chronic and accelerated phases of this condition,” said Professor Longson.

NICE has recommended two new treatment options for patients with multiple myeloma.

Celgene’s Thalidomide has been recommended as a first-line treatment in people where high-dose chemotherapy with stem cell transplantation is considered inappropriate. If the patient is intolerable to Thalidomide, Janssen’s Velcade (Bortezomib) is then recommended.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE, claimed that the new regimens have been shown to be “more effective at delaying disease progression and improving patients’ life expectancy.”

Multiple myeloma is a rare type of cancer that develops from cells in the bone marrow. Almost 4,000 cases are diagnosed every year in the UK.

Mike Hobday, Head of Policy at Macmillan Cancer Support, stated: “It is great news that NICE has recommended these drugs for cancer patients with multiple myeloma and that their needs have been listened to.

“For too long patients with less common cancers, such as multiple myeloma have lost out on receiving the vital medicines they need on the NHS.

“This decision recognises the need to fund drugs that improve the quality and length of life for cancer patients with rarer cancers, which is a key step to improving better access to treatments for all.”