It is looking unlikely that the reach of Celgene’s Vidaza as a treatment for the blood cancer acute myeloid leukaemia will be extended on the NHS in line with its expanded regulatory approval in Europe.

The National Institute for Health and Care Excellence has now published draft recommendations rejecting the drug 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). 

Vidaza (azacitidine) is already recommended by the Institute for adults not eligible for 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.

According to the firm, Vidaza “has the potential to provide a vital life-line to eligible people with AML”, having shown in clinical trials a median improvement in survival of 3.8 months (10.4 vs 6.5 months) compared with conventional care regimens.

“Azacitidine (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,” said Zack Pemberton-Whiteley, head of Campaigns and Advocacy at Leukaemia CARE.

However, in the appraisal consultation document, NICE’s committee concludes that the drug “should not be considered a step change” in the treatment of AML and that there was “a high degree of uncertainty about its clinical effectiveness relative to current conventional chemotherapy treatments”.

Moreover, while the company’s own modelling generated an incremental cost-effectiveness ratio for the drug of around £20,000 per QALY, NICE’s expert review group felt the figure would be closer to £240,000 per QALY, and thus well outside the bounds of what is normally considered value for money for the NHS.

Celgene said is it committed to working with NICE throughout the appraisal process.