NICE has promised to re-appraise the use of Vidaza (azacitidine) in patients with higher-risk myelodysplastic syndrome (MDS) – a group of debilitating bone marrow diseases.
The decision has come following months of campaigning on the part of MDS charities and patient groups.
The NICE appeal panel has requested that the appraisal committee reconsider the guidance it originally issued on azacitidine, taking account of low-dose chemotherapy as well as best supportive care as comparisons for cost-effectiveness. The committee will also examine data on quality of life provided by the MDS UK Patient Support Group.
David Hall, Chairman of the MDS UK Patient Support Group, 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.”
NICE recommended that azacitidine should not be provided through the NHS on cost-effectiveness grounds in March 2010, but said that it recognised the drug was clinically effective and that it should be seen as a life-extending end-of-life treatment. In June the decision was appealed by eight organisations, including three patient groups and four healthcare professional bodies.
MDS which can lead to complications such as recurrent or life-threatening infections or bleeding and can progress to acute myelogenous leukemia (AML), which has 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 has been shown to improve patients’ quality of life substantially, reduce dependence on blood transfusions and is less toxic than chemotherapy.
The NICE announcement coincided with the launch of a Government cancer treatments fund of £50 million to be available to patients until the full promised £200 million fund comes into effect from April 2011.