NICE rejects azacitidine again in their latest draft guidance

In preliminary draft guidance issued for public consultation NICE has not recommended azacitidine (Vidaza, Celgene) as a treatment option for people who have the following conditions, and are not eligible for haematopoietic stem cell transplantation: intermediate-2 and high-risk myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia.

The committee decided that although azacitadine was shown to be clinically effective, the benefits to patients could not justify the high cost of the drug.

This draft guidance has been issued for consultation. 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 the manufacturer of azacitidine (Celgene), 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.

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.

NICE Chief Executive, Sir Andrew Dillon said: “The independent Appraisal Committee reconsidered 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.


  1. The guidance is available here.
  2. According to the manufacturer’s estimates, azacitidine costs approximately £45,000 per patient.
  3. The manufacturer of azacitidine has agreed 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.
  4. The committee agreed that azacitidine did fit the criteria to be 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.
  5. The committee agreed that the most plausible ICER for azacitidine in the overall patient population was approximately £59,000 per QALY gained.

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