The cost regulator for National Health Service therapies in England and Wales has closed the door to Celgene’s Vidaza as a treatment for a group of blood disorders on grounds that it is too expensive.

The National Institute for Health and Clinical Excellence has this morning published a Final Appraisal Determination rejecting Vidaza (azacitidine) for myelodysplastic syndromes (MDS), chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML), after calculating the drug’s incremental cost to be £63,000 per quality adjusted life year.

MDS encompasses a group of incurable blood disorders affecting the bone marrow, which can develop into CMML or, in 30% of cases, AML, for which the prognosis is very poor as patients can die in just six to 12 months following diagnosis. Treatment options are limited, with strategies currently focused on symptom management as opposed to treating the disease itself.

Vidaza - the first in a new class of epigenetic therapies that focus on genetic errors behind disease - is thought to works by reviving the natural mechanism of cells that keeps abnormal growth under control 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.

A study published in The Lancet Oncology showed that the median overall survival for higher-risk MDS patients given Vidaza was 24.5 months compared to 15 months for those receiving conventional care, and that, at two years, the survival rate was 50.8%, almost twice that of patients on standard care (26.2%).

News of its success in clinical trials and subsequent European approval in March last year provided a new ray of hope to the 700 or so patients (a year) who could be eligible for treatment with Vidaza, but the Institute’s decision means that they will only be able to access the drug if they pay for it privately as it will not be reimbursed on the NHS.

“We are extremely disappointed NICE has rejected the only drug available proven to give patients with myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia more time and a better quality of life,” commented Mike Hobday, Head of Campaigns at Macmillan Cancer Support.

'Failing patients'
He slammed the current system for “failing” people with rarer cancers and noted that Vidaza is one of several drugs for rarer cancers to be rejected by NICE in the past year, and he called for “a more flexible approach – one that doesn’t rule you out if you have the wrong kind of cancer”.

The consultation period for the FAD is now open until March 18. Macmillan says it will not be appealing the guidance, but Celgene has announced that it will fight the Institute’s decision.

“The appeal Celgene will file will detail that Vidaza as a treatment for MDS meets not only the end of life criteria (as set out by the Department of Health), but also the innovation criteria (as set out by NICE) both of which were designed to increased access to innovative medicines in areas of unmet need”, the company said, and added that drug is currently approved and reimbursed in more than 30 countries around the world.