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NICE unable to recommend bone marrow disease drug

Newspage: microscope 3NICE is disappointed to be unable to recommend the use of azacitidine (Vidaza) for the treatment of myelodysplastic syndromes (MDS), a group of rare bone marrow disorders.

There are around 700 patients in England and Wales with myelodysplastic syndrome. Currently, they are offered supportive care which may include blood transfusions.

The manufacturers of the drug, Celgene, have agreed a patient access scheme with the Department of Health which has helped to reduce the cost of the drug to the NHS. This was taken into consideration when producing the final draft guidance.

However, it has been calculated that making azacitidine available in the general patient population would still cost £63,000 per quality-adjusted life year (QALY) gained.

NICE uses QALYs when making decisions as they are an internationally recognised method of comparing different drugs and measuring their clinical effectiveness. It helps to give an idea of how many extra months or years of life of a reasonable quality a person might gain as a result of treatment.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE said: “Azacitidine is the first drug that has been developed specifically for treating MDS. It is not a cure, but could potentially prolong the life of people with these conditions by around nine months longer than standard treatment.

“We are disappointed not to be able to recommend this drug. The independent Appraisal Committee considered all published evidence on the effectiveness 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.”

“The committee agreed that azacitidine did fit the criteria to be considered under the supplementary advice for end of life medicines; however, even when both the patient access scheme and the end of life criteria were incorporated the cost of the drug, relative to its benefit was too great.”

4 March 2010

This page was last updated: 13 April 2010

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Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.