NICE says no to bone marrow disease drug

NICE has decided that azacitidine will not be available through the NHS

A drug for treating rare blood cancers will not be made available through the NHS in England and Wales.

The National Institute for Health and Clinical Excellence (NICE) has decided not to recommend azacitidine or Vidaza for treating myelodysplastic syndromes because it is too costly.

Cancer charities say they are angered and disappointed by the decision.

Around four in 100,000 people in the UK suffer from MDS - a debilitating bone marrow disease.

Myelodysplastic syndromes (MDS) mean that the bone marrow does not produce enough of one or more types of blood cells.

Most MDS patients have to rely on frequent blood transfusions to manage anaemia and extreme fatigue.

The average survival of patients with MDS is about 20 months. Nearly a third of patients progress to acute myeloid leukaemia.

NICE says that the majority of patients with MDS receive the best care in current clinical practice.

Not a cure

Dr Carole Longson is health technology evaluation centre director at NICE. She said that azacitidine is not a cure for MDS but could potentially prolong the lives of people with these conditions by around nine months longer than standard treatment.

However she stated: "The Appraisal Committee concluded that relative to the benefits, the price the NHS is being asked to pay for azacitidine is still too high for it to be recommended as a cost effective use of NHS resources."

The manufacturer of the drug, Celgene UK, has announced that it plans to appeal NICE's draft guidance ruling.

According to the manufacturer's estimates, azacitidine costs approximately £45,000 per patient.

David Hall is an MDS patient. He is also chairman of the MDS UK Patient Support Group, which is supported by pharmaceutical companies, including Celgene.

He said the decision was a huge blow to MDS patients.

"A total of only 700 patients a year in England and Wales would require treatment with azacitidine so we do not believe that providing this life-extending treatment would make a huge impact on the NHS budget," he said.

Source BBC