Cost issues have prevented a bone marrow disease drug from becoming available to English and Welsh patients, it emerged on March 4th 2010.

The drug in question is azacitidine – which is marketed as Vidaza – and it has been approved by the US Food and Drug Administration (FDA) for use in treating patients with MDS (myelodysplastic syndromes) in the US, as well as in 30 or so other countries.

However, as far as NICE – the National Institute for Health and Clinical Excellence – is concerned, its cost prohibits similar approval for use by the National Health Service.

MDS: Bone Marrow Disease

MDS is a bone marrow disease that affects a few hundred people in the UK (with four cases per 100,000 people). Those that have it experience a drop in the bone marrow’s ability to produce blood cells and, therefore, require a programme of blood transfusions to be set up.

MDS patients can expect to live for approximately 20 months, on average. Around thirty per cent subsequently develop a type of leukaemia.

Vidaza: MDS Treatment

Vidaza is produced by a UK firm, Celgene UK, which has stated that it will contest NICE’s verdict. Guide figures produced by this firm put the per-patient treatment cost at £45,000.

“The appeal Celgene will file will detail that Vidaza as a treatment for MDS meets not only the end of life criteria (as set out by the Department of Health), but also the innovation criteria (as set out by NICE) both of which were designed to increased access to innovative medicines in areas of unmet need”, it said in a statement.

A NICE representative told the BBC that Vidaza could not cure MDS, but it had the ability to extent patients’ lives. Nonetheless, said Doctor Carole Larson: “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 news has been received badly by cancer charities.

“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”, David Hall – representing the MDS UK Patient Support Group – stated.