NICE rejects drug for rare pre-cancerous conditions

In collaboration with Adfero

A drug for treating a group of conditions that affect the bone marrow is unlikely to be made available on the NHS in England and Wales after the body which provides independent advice on treatments said that it is unlikely to be cost-effective.

Azacitidine (brand name Vidaza) is the first drug that is specifically designed to treat myelodysplastic syndromes (MDS) - a group of conditions in which the bone marrow does not produce enough healthy blood cells.

The syndromes are not cancer, but they can sometimes develop into leukaemia.

Although azacitidine is not a cure for MDS, research suggests that it may prolong the lives of patients with the conditions by around nine months longer than standard treatment.

The National Institute for Health and Clinical Excellence (NICE) has now published final draft guidance in which it recommends that azacitidine should not be offered to people with myelodysplastic syndromes who are ineligible for stem cell transplants.

Dr Carole Longson, director of NICE's Health Technology Evaluation Centre, said that the institute was disappointed not to recommend the drug.

She said: "The independent Appraisal Committee considered all published evidence on the effectiveness of azacitidine and the cost, including the proposed 'patient access scheme'. 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."

Dr Longson noted that the drug was also considered under the supplementary advice for end of life medicines.

"However, the magnitude of additional weight that would need to be assigned to the original QALY for the cost-effectiveness of the drug to fall within the current threshold range would be too great, even when the patient access scheme was incorporated," she explained.

This decision is now open to appeal. It is widely expected that the manufacturers of azacitidine, Celgene, will be responding with an appeal. If this is not successful, NICE's decision will be published in final guidance in May 2010.

Professor Peter Johnson, Cancer Research UK's chief clinician, said: "Cancer Research UK is extremely disappointed that NICE has failed to approve azacitidine for use in patients with myelodysplastic syndrome in the NHS. This decision goes against the progress we are making and leaves these patients without any real options for treatment.

"We know NICE has heard from clinical experts about the benefits of this drug - not only in slowing down the disease, but also in improving quality of life.

"Myelodysplastic syndrome is a pre-cancerous condition that can often progress to leukaemia. Around 700 people could be suitable for treatment with azacitidine so the overall impact on the NHS budget would be small.

"Increasingly, NICE is returning negative decisions for cancer drugs where only a small number of people will require treatment and these patients have a short life expectancy. We want the Department of Health to consider whether NICE is indeed the appropriate place to make decisions about these sorts of drugs."