Cost regulators are sticking with a decision to bar some patients with acute myeloid leukaemia from routine NHS access to Celgene's Vidaza.

The National Institute for Health and Care Excellence has rejected the drug's use as an option to treat AML in patients aged 65 years or older with more than 30 percent bone marrow blasts who are not eligible for haematopoietic stem cell transplantation (HSCT).

In final draft guidance, NICE described a high degree of uncertainty about Vidaza's (azacitidine) clinical effectiveness relative to current treatments in the target patient population. The incremental cost-effectiveness ratio was calculated to be around £240,000 per QALY, and therefore well outside the bounds of what is normally considered a cost-effective use of NHS resources.

Zack Pemberton-Whiteley, head of Campaigns and Advocacy at Leukaemia CARE, previously said that Vidaza "could provide a tolerable treatment option for patients who, on average, live just two months post diagnosis if they do not receive active treatment - it is vitally important they are able to benefit from a medicine which can help extend their time with family and loved ones, whilst not compromising quality of life."

Vidaza is already recommended by NICE for adults not eligible for haematopoietic stem cell transplantation (HSCT) with 20 - 30 percent blasts and multilineage dysplasia, as well as for those with intermediate- and high-risk myelodysplastic syndromes or chronic myelomonocytic leukaemia with 10 - 29 percent marrow blasts without myeloproliferative disorder.

Its label was expanded in Europe back in November to include AML patients who have more than 30 percent myeloblasts, on the back of clinical data showing that median overall survival was 10.4 months for patients receiving the drug versus 6.5 months for those taking conventional treatment regimens, bringing new hope to those unable take the HSCT route.