The appeal Celgene expects to file will detail that Vidaza as a treatment for MDS meets not only the end of life criteria (as set out by the Department of Health), but also the innovation criteria (as set out by NICE) both of which were designed to increased access to innovative medicines in areas of unmet need.
Vidaza is currently licensed for the treatment of patients not eligible for hematopoietic stem cell transplantation with intermediate-2 and high-risk MDS, chronic myelomonocytic leukaemia (CMML) with 10 to 29 % marrow blasts without myeloproliferative disorder and acute myeloid leukaemia (AML) with 20-30 percent Blasts and Multi-Lineage Dysplasia.
VIDAZA is currently approved and reimbursed for patients with higher-risk MDS, AML and CMML in more than 30 countries around the world.
Reportedly, a study published in ‘The Lancet Oncology’ demonstrated that the median overall survival for higher-risk MDS patients receiving VIDAZA was 24.5 months compared to 15 months for patients receiving conventional care regimens such as supportive care or chemotherapy a difference of 9.5 months. The study also showed that at two years, the survival rate for patients receiving VIDAZA was 50.8%, nearly double that of patients receiving conventional care regimens (26.2%).
Sam Pearce, general manager of Celgene UK, said: “There is no higher priority for Celgene than to ensure that patients are able to access our life-extending therapies for rare blood cancers. Through this appeal we are committed to working tirelessly to reach a positive outcome for patients.”