In preliminary draft guidance issued for public consultation, NICE, the UK National Institute for Health and Clinical Excellence, is the independent organisation responsible for providing national guidance on the promotion of good health and the prevention and treatment of ill health, has not recommended azacitidine (Vidaza?, Celgene) as a treatment option for people who are diagnosed with intermediate-2 and high-risk myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia but are not eligible for hematopoietic stem cell transplantation. This draft guidance has been issued for consultation.

While azacitidine, which is indicated for treatment of patients with the French-American-British (FAB) myelodysplastic syndrome subtypes refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL), was shown to be clinically effective, the committee decided that the benefits to patients could not justify the high cost of the drug, estimated to be approximately ?45,000 per patient.

NICE has not yet issued final guidance to the NHS. Publication of this second appraisal consultation document follows the return of the appraisal to the independent Appraisal Committee after appeals against the Institute’s final appraisal determination were partially upheld. The appeals were received from Celgene, the manufacturer of azacitidine, a joint appeal from the Royal College of Pathologists and British Society of Haematology, a joint appeal from the Leukaemia Society, Rarer Cancer Forum and MDS UK Patient Support Group and one from the Royal College of Physicians Medical Oncology Joint Special Committee.

Celgene has agreed to a patient access scheme with the Department of Health, in which azacitidine for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia will be available with a 7% reduction in the acquisition cost.

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The Appeal Panel requested that the Appraisal Committee reconsider the guidance, taking account of a wider range of comparators than originally considered. The Appeal Panel also suggested that the Appraisal Committee examine data on quality of life submitted by MDS UK.

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NICE Chief Executive, Sir Andrew Dillon said: ?The independent Appraisal Committeereconsidered the evidence on the effectiveness of azacitidine, when compared with best supportive care and low-dose chemotherapy. They also considered the additional data submitted by MDS UK on quality of life. However, the Committee again concluded that the drug could not be recommended as a cost effective use of NHS resources. This is a very expensive drug, even with the small discount offered by the manufacturer.?

?Although we are very disappointed not to be able to recommend this treatment, we have assessed it fairly and on precisely the same basis as other drugs used for rarer conditions.?

Myelodysplastic syndromes (MDS) are a group of bone marrow disorders, where the marrow doesn’t produce enough of one or more types of blood cells. The majority of patients with MDS receive best supportive care in current clinical practice and some patients receive low dose chemotherapy. There are approximately 700 patients with MDS in England and Wales.

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