The National Institute for Health and Clinical Excellence is sticking with its decision that Celgene’s Vidaza is too expensive for use on the National Health Service to treat patients with myelodysplastic syndromes (MDS).

The cost watchdog has republished preliminary guidance again rejecting Vidaza (azacitidine) as a treatment for intermediate-2 and high-risk myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia, in patients unable to undergo stem cell transplantation.

A stream of partially successful appeals from a number of parties - including the Royal College of Pathologists the Leukaemia Society and MDS UK Patient Support Group - objecting to NICE’s previous draft guidance on Vidaza forced the Institute to have another look at the drug, taking into account more comparators and quality of life data from MDS UK.

But in a huge blow to the MDS community in England and Wales, despite reconsidering the evidence the committee concluded that while the drug was indeed shown to be clinically effective, its benefits could not justify its high cost – which comes in at around £45,000 per patient.

Even taking into account a patient access scheme, under which Celgene would offer Vidaza at a 7% reduction in cost, and that the drug met the criteria for appraising so-called end-of-life treatments, the committee calculated its most plausible incremental cost to be around £59,000 per QALY gained, and so well above the bar of what is normally considered acceptable for NHS use.

The news will be devastating for many patients with MDS, particularly as the prognosis can be very poor and treatment options are limited, largely aiming to control the symptoms rather than address their cause, and given that Vidaza is the first in a new class of epigenetic therapies that actually focus on the genetic errors behind disease.

It is thought that the drug encourages the natural mechanism of cells that keeps abnormal growth in check and, in clinical trials, Vidaza extended median survival in higher-risk patients from 15 months to over two years when compared to conventional care regimens.

But commenting on the Institute’s decision, its chief executive Sir Andrew Dillon stressed that the drug is very expensive, even with the small discount offered by the Celgene. “Although we are very disappointed not to be able to recommend this treatment, we have assessed it fairly and on precisely the same basis as other drugs used for rarer conditions”, he added, defending the decision.