Several UK cancer patient support groups have voiced their anger at the National Institute for Health and Clinical Excellence (NICE)’s Final Appraisal Determination (FAD) that Vidaza (azacitidine) will not be recommended to treat patients with intermediate-2 or high risk myelodysplastic syndromes (MDS), nor for patients with forms of chronic myelomonocytic leukaemia or acute myeloid leukaemia.
MDS affects around four in 100,000 people in the UK and can lead to recurrent or life-threatening infections or bleeding. The average survival rate is 20 months. Most patients need frequent blood transfusions and around a third will go on to develop acute myeloid leukaemia (AML), an aggressive and resistant form of leukaemia with survival rates of only a few months.
The UK health watchdog’s decision not to allow the drug’s use on the NHS was made on cost-effective grounds. It is recommended that patients receive at least six cycles of treatment, costing around £17,000.
David Hall, chairman of the MDS UK Patient Support Group and an MDS patient said: “This is a huge blow to MDS patients, particularly those with the high risk forms of these diseases for whom the outlook is often bleak. Azacitidine is the only licensed drug available specifically to treat MDS and has been proven not only to slow the progress of the disease but also vastly improve patients’ quality of life by freeing them from repeated cycles of blood transfusions.
“A total of only 700 patients a year in England and Wales would require treatment with azacitidine so we do not believe that providing this life-extending treatment would make a huge impact on the NHS budget.”
Operating director of the Myelodysplastic Syndromes Foundation, Kathy Heptinstall, points out that: “This negative appraisal of Vidaza from NICE denies MDS and AML patients access to the only drug proven to prolong their lives.”
Celgene, the drug’s manufacturer, plans to appeal the negative recommendation.
“There is no higher priority for Celgene than to ensure that patients are able to access our life-extending therapies for rare blood cancers,” said Sam Pearce, general manager of Celgene UK. “Through this appeal we are committed to working tirelessly to reach a positive outcome for patients.”
Celgene’s appeal will reference that Vidaza meets both the Department of Health (DH)’s end of life criteria and NICE’s innovation criteria – designed to increase access to innovative medicines in areas of unmet medical need.
Formal consultees have 15 working days (until 18 March 2010) to lodge an appeal against. Subject to any such appeal(s), the FAD may be used as the basis for NICE’s guidance on the use of the appraised technology in the NHS in England and Wales.