Celgene’s anticancer drug Vidaza has been turned down by the National Institute for Health and Clinical Excellence for treating blood cancers on the basis that it would not represent a cost-effective use of National Health Service resources.

Vidaza (azacitidine) - the first in a new class of epigenetic therapies that focus on genetic errors behind disease - was approved by UK regulators back in March to treat myelodysplastic syndromes, including the subgroups chronic myelomonocytic leukaemia (CML) and acute myeloid leukaemia (AML).

MDS encompasses a group of incurable blood cancers that occur in immature blood cells, for which treatment to date has been primarily focused on symptom management. Around 2,000 patients are diagnosed with MDS every year, and, according to the MDS Foundation, in 30% of cases the disease will progress to AML, for which the prognosis is very poor as patients can die in just six to 12 months.

It is thought that the drug works by reviving the natural mechanism of cells that keeps a handle on any abnormal growth and, in clinical trials, was shown to significantly extended median survival in higher-risk patients from 15 months to over two years when compared to conventional care regimens.

Clinically effective but too costly
But despite agreeing that Vidaza is indeed clinically effective within its licensed indications, NICE’s Committee felt that treatment with the drug is just too costly for use on the health service. Although it was unable to determine a final cost-effectiveness estimate, the Committee concluded that the ICER would be significantly higher than the manufacturer’s base case scenario of £66,000 per QALY (quality-adjusted life year), and so could not endorse its use, even when taking into account new guidance for appraising end-of-life drugs.

David R Hall, Chairman of the MDS UK Patient Support Group, said patients will be "devastated" by NICE's preliminary recommendations. "Celgene’s VIDAZA has brought a ray of hope to MDS sufferers, particularly those with the higher risk conditions" as it "replaces the uncomfortable and tedious blood transfusion regime" and extends expected survival, he explained.

"Placing an arbitrary value on human life is presumptuous in any situation," he said, and added: "When suffering in old age, with premature death are the criteria, our society needs to look critically at the avenues through which public funds are dispensed".

The Committee’s conclusions could still be contested, however, and the closing date for comments is August 24 August in time for a Second Appraisal Committee meeting on September 3.