The National Institute for Health and Clinical Excellence (NICE) is currently appraising the use of azacitidine (Vidaza, Celgene) for myelodysplastic syndromes. In the final draft guidance, published today (4 March 2010), NICE has not recommended azacitidine as a treatment option for people who have the following conditions, and are not eligible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
- acute myeloid leukaemia
In line with the NICE technology appraisals process this draft guidance is now with consultees, who have the opportunity to appeal against the proposed guidance. NICE has not yet issued final guidance to the NHS. Final guidance is expected to be published in May 2010.
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.
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 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.
"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."