Celgene UK will appeal NICE’s decision against the use of Vidaza (azacitidine) in patients with higher-risk myelodysplastic syndrome (MDS), a rare type of blood cancer.
NICE has concluded that the drug does not represent a good use of NHS funds, though it is reimbursed in this indication in most other European countries.
A group of UK cancer patient support organisations have also expressed their disappointed and anger and have set up a petition against the decision.
Celgene will argue that Vidaza as a treatment for MDS meets not only the end of life criteria (set out by the Department of Health), but also the innovation criteria (set out by NICE).
“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.”
MDS is a rare cancer with high unmet need, limited treatment options and a poor prognosis. Though chemotherapy is the conventional care regimen for these patients, NICE’s decision will mean that supportive care is the only alternative option.
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 supportive care or chemotherapy.
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%).
Vidaza is currently licensed for the treatment of patients not eligible for hematopoietic stem cell transplantation with intermediate-2 and high-risk MDS. It is also licensed for chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML).
The drug is currently approved and reimbursed for patients with higher-risk MDS, AML and CMML in more than 30 countries around the world.
David Hall, Chairman of the MDS UK Patient Support Group, 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.”
The petition calling on the Prime Minister to ensure that MDS care in the UK is comparable with that in other European countries has so far attracted over 300 signatures. It is available at http://www.ukmdsforum.org/petition.php.