London, 26 July 2010 – The MDS UK Patient Support Group has welcomed the decision announced today by the National Institute for Health and Clinical Excellence (NICE) that it will re-appraise the use of Vidaza (azacitidine)in patients with higher-risk myelodysplastic syndrome (MDS)- a group of debilitating bone marrow diseases.
Today’s decision by a NICE Appeal Panel requests that the organisation’s Appraisal Committee reconsider the guidance it originally issued on azacitidine, this time taking account of low-dose chemotherapy as well as best supportive care as comparisons for cost-effectiveness. The Appeal Panel also requests that the Committee examine the data on quality of life provided by the MDS UK Patient Support Group.

David Hall, Chairman of the MDS UK Patient Support Group and MDS patient said, “We welcome this decision by NICE to re-appraise azacitidine and believe it has been brought about by the weight of patient, expert and clinical opinion behind the arguments put to the Appeal Panel. 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.”

In March 2010, NICE recommended that azacitidine should not be provided through the NHS on cost-effectiveness grounds while recognising that it was clinically effective and that it should be seen as a life-extending end-of-life treatment. In June this decision was appealed by eight organisations, including three patient groups and four healthcare professional bodies.
Approximately four in 100,000 people in the UK have MDS which can lead to complications such as recurrent or life-threatening infections or bleeding and which can progress to an aggressive form of leukaemia with an average survival period of a few months only. Azacitidine is the only licensed medicine proven to prolong survival for people with higher risk MDS and could be suitable for 700 patients per year in England and Wales. It has been shown to improve patients’ quality of life substantially, reduce dependence on blood transfusions and is less toxic than chemotherapy.
The MDS UK Patient Support Group awaits more details about the £200 million Cancer Drug Fund promised by the Government with interest. However it will come too late for the many MDS patients who have anxiously been awaiting the outcome of this very long NICE appraisal.

David Hall said, “We are pleased to see this government commitment to making all clinically proven cancer treatments available to patients through the NHS. We hope that the Cancer Drug Fund will be set up in such a way as to ensure that funding follows clinicians’ decisions as, in our experience, clinicians are best placed to advise on the most appropriate treatment for MDS patients.”
The NICE announcement can be found at the NICE website.
Organisations which appealed the NICE decision: MDS UK Patient Support Group, the Rarer Cancer Forum, the Leukaemia Society, the National Cancer Research Institute Haematological Oncology Clinical Studies Group, the Royal College of Physicians’ Medical Oncology Joint Special Committee, the Royal College of Pathologists, the British Society for Haematology and Celgene Ltd.

Free donations by shopping