MDS patients in the UK will be celebrating a dramatic u-turn by the National Institute for Health and Clinical Excellence on the use of Celgene's Vidaza on the National Health Service.

The cost regulator for treatments on the National Health Service in England and Wales has issued draft guidance recommending the use of Vidaza (azacitidine) for patients with myelodysplastic syndromes, specifically those with intermediate-2 and high-risk forms of the condition, chronic myelomonocytic leukaemia and acute myeloid leukaemia who are not eligible for haematopoietic stem cell transplantation.

The decision comes at the end of a long-running saga which involved a number of parties - including the Royal College of Pathologists, the Leukaemia Society and MDS UK Patient Support Group - battling against NICE's original stance that the drug is too expensive for NHS use.

Following a stream of partially successful appeals, the Institute was forced to take another look at the evidence supporting Vidaza's use, taking into account more comparators and quality of life data from MDS UK. But in November there was much disappointment when NICE republished draft guidance turning down the drug again despite its re-analysis. 

However, in a last-ditch attempt Celgene then dropped the price of Vidaza and resubmitted its proposed patient access scheme, which helped persuade the NICE that it is indeed a cost-effective use of NHS resources.

While a spokeswoman for the Institute could not divulge any further details on the revised patient access scheme, which is being kept under wraps by the drugmaker, she did confirm to PharmaTimes UK News that Celgene's discount brings the medicines under the £50,000 per QALY threshold, thereby opening the door to its use on the health service.

While Vidaza does not offer a cure for MDS, it has the potential to extend patients’ lives by an average of nine months, and the Institute said it is pleased to be able to recommend the drug's use in hospitals throughout England and Wales.

News welcomed

Others also lined up to welcome the news. MP John Leech said he is "delighted" with NICE's decision. "This is great news for patients with MDS and their families", he said, and congratulated all those who "have fought hard for this victory”.

"This is the best thing that has happened to MDS patients in the UK for a long time," added Ghulam Mufti, Consultant Haematologist at King’s College Hospital, London, and longtime campaigner for MDS patients. "UK patients will finally have access to the same standard of care as MDS patients in the rest of Europe and in the USA. Fabulous news.”

And MDS UK Patient Support Group deputy chairman Rodney Taylor noted that Vidaza is "the only specific treatment for MDS that improves quality of life, prolongs survival and delays disease progression," and said "it is great news that many more patients will now be able to benefit from it".

On the downside, the group voiced its concern that access to the drug is still denied to MDS patients in Scotland, as Vidaza has yet to be re-submitted to the Scottish Medicines Consortium following its rejection in April last year.