Patients in the UK with myelodysplastic syndromes will be celebrating news that the National Institute for Health and Clinical Excellence has been ordered to reassess Celgene’s Vidaza for the National Health Service.

An Appeal Board has agreed with certain objections voiced in appeals by Celgene and a whole host of other bodies - including Royal Colleges and support group MDS UK - against a ruling by the cost watchdog to bar patients with MDS, chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML) from access to Vidaza (azacitidine) on the NHS.

MDS is a group of incurable blood disorders affecting the bone marrow that can develop into CMML or, in 30% of cases, AML, which leaves patients with a very poor prognosis of having just six to 12 months to live following diagnosis. Treatment options remain limited, and current strategies are very much focused on symptom management as opposed to disease treatment.

The UK approval of Vidaza in March sparked much excitement in the MDS community as the first in a new class of epigenetic therapies focusing on 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, extended median survival in higher-risk patients from 15 months to over two years when compared to conventional care regimens.

Further evidence of its potential was shown by a study published in The Lancet Oncology demonstrating 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%).

However, NICE published a Final Appraisal Determination rejecting Vidaza for MDS, CMML and AML after calculating the drug’s incremental cost to be £63,000 per quality adjusted life year, and ruling that this is simply too expensive for NHS use.

'Perverse' ruling
But in its economic modelling the Institute failed to accept low-dose chemotherapy as a comparator, which, in its appeal, Celgene argued was “perverse” given that this form of therapy is in widespread use by the NHS for MDS.

The Appeal Panel agreed, and, consequently, ordered the NICE’s Appraisal Committee to re-assess Vidaza, taking into account both best supportive care and low-dose chemotherapy. It also requested that the Committee take into consideration quality of life data provided by the MDS UK Patient Support Group.

David Hall, Chairman of the MDS UK Patient Support Group has welcomed the decision to reappraise Vidaza. “We only wish that the Appraisal Committee had considered these points in its initial appraisal and made this life-extending therapy available to patients from early this year. Instead patients are still waiting to see if they will be thrown a lifeline.”

And Sam Pearce, General Manager of Celgene UK and Ireland, told PharmaTimes UK News that the decision "is good news for MDS patients and their families" and that the company is "delighted that the NICE appeal body has asked the appraisal committee to review their original decision which would have denied MDS patients access to Vidaza - the only treatment that is proven to significantly extend life".

"The NICE assessment process poses real challenges for medicines that are used to treat rare conditions, but we look forward to working with NICE to ensure MDS patients have access to this vital treatment as soon as possible", he added.