The National Institute for Health and Clinical Excellence (NICE) has issued final guidance recommending azacitidine (Vidaza ®) as a treatment option for patients with myelodysplastic syndromes (MDS) who are not eligible for haematopoietic stem cell transplantation. The decision, which was issued on March 23, represents an about turn for NICE who in November 2010 refused to recommend use of azacitidine on the NHS claiming that the drug was not cost effective even under a patient access scheme where Celgene offered the drug at a reduced price.

Celgene has now increased its contribution under the patient access scheme but agreed with the Department of Health that the figure should remain confidential.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE, said: "Azacitidine is the first drug that has been developed specifically for treating myelodysplastic syndromes. It is not a cure but it does have the potential to extend patients' lives by an average of nine months. It is a very expensive drug, but the manufacturers have submitted a patient access scheme where the cost will be reduced. We are therefore very pleased to be able to recommend azacitidine as a cost effective use of NHS resources."

Myelodysplastic syndromes (MDS) are a group of disorders in which stem cells in the bone marrow do not mature properly into healthy blood cells. Instead, immature blood cells (blasts) accumulate that die in the bone marrow or soon after they enter the blood stream. In approximately one third of patients the condition progresses to acute myeloid leukemia (AML).

Azacitidine, the first drug that has been developed specifically for treating MDS, is an anti-neoplastic pyrimidine nucleoside analog that exerts a cytotoxic effect on rapidly dividing cells, including cancerous cells, and may help restore normal function to genes controlling proper cellular differentiation and proliferation. The FDA approved azacitidine based on an open label controlled trial that randomized 191 patients with MDS to receive either subcutaneous azacitidine plus best supportive care (n=99) or supportive care alone (n=92). The median overall survival for patients treated with azacitidine in the study was 24.5 months compared to 15 months for those allocated conventional care regimens (P value = 0.0001).

Source: NICE