The MDS UK Patient Support Group says it has lodged an appeal against the negative recommendation issued earlier this month by the National Institute for Health and Clinical Excellence (NICE) for the use of Celgene's Vidaza (azacitidine) in patients with higher-risk myelodysplastic syndrome (MDS; The Pharma Letter March 4). The Leukaemia Society and the Rarer Cancers Forum are also appealing the NICE guidance.
Approximately four in 100,000 people in the UK have MDS, a group of debilitating bone marrow diseases that lead to complications such as recurrent or life-threatening infections or bleeding. Most MDS patients have to rely on frequent blood transfusions to manage anemia and extreme fatigue. While the average survival of patients with MDS is about 20months, nearly a third (30%) progress to acute myeloid leukaemia (AML), a very aggressive and resistant form of leukemia with an average survival period of a few months only.
Cost per patient is $45,000
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