NICE recommends azacitidine for myelodysplastic syndromes

In collaboration with Adfero

The National Institute for Health and Clinical Excellence (NICE) has approved a drug called azacitidine (brand name Vidaza) for people with myelodysplastic syndromes (MDS) - a group of conditions that affect the bone marrow.

The institute, which provides the NHS with independent advice on treatments, published its final guidance on this drug earlier this week. This decision follows an initial assessment in which they ruled that the drug was not cost-effective.

But a patient access scheme agreed presented to the Department of Health by the drug's manufacturer has enabled NICE to reassess its earlier decision.

Azacitidine is the first drug that is specifically designed to treat MDS, in which a patient's bone marrow fails to produce enough healthy blood cells.

It is now recommended for certain patients with intermediate-2 and high-risk myelodysplastic syndromes, chronic myelomonocytic leukaemia or acute myeloid leukaemia.

In order to qualify for treatment with azacitidine, patients must be ineligible for a stem cell transplant.

While azacitidine does not provide a cure for these conditions, clinical trials have shown that it may extend patients' life expectancy by an average of nine months.

The decision could benefit about 700 patients in England and Wales who have the type of MDS that the drug is licensed to treat.

Dr Carole Longson, director of the NICE Health Technology Evaluation Centre, said the institute was pleased to be able to recommend the new treatment.

She revealed: "During consultation on the draft recommendations, the manufacturer of azacitidine offered to provide the drug at a reduced price.

"Azacitidine is an expensive drug, and this discount enabled us to recommend it as a cost-effective use of resources on the NHS."

Hilary Tovey, Cancer Research UK's policy manager, said: "We're pleased that NICE has reversed its previous decision not to recommend this drug. This is good news for people with myelodysplastic syndromes who might now be able to benefit from this treatment."