The Scottish Medicines Consortium (SMC) has approved the use of Celgene's Vidaza (azacitidine) in patients with advanced myelodysplastic syndromes (MDS).
The Consortium's decision takes into account the benefits of a new patient access scheme (PAS) for Vidaza, which is the only drug available in Europe for MDS, a rare but debilitating disease which affects around 2,150 people in the UK each year.
The average survival of high-risk MDS patients is between 0.4 and 1.2 years, and about 20%-30% of patients progress to acute myeloid leukaemia which, when it occurs in the MDS setting, is almost always lethal.
Until now, many MDS suffers have been reliant on frequent blood transfusions, which provide only short-term symptomatic improvements.
Vidaza's approval has been welcomed by Dr Paul Macellef-Eynaud, consultant haematologist at NHS Ayrshire and Arran, who said he hoped that by providing patients with access to the drug, the rate of survival and quality of life for many MDS suffers would be improved.
"Individuals with advanced MDS can suffer from bleeding, infection and severe tiredness, and there is a high risk of transformation to acute leukaemia, at which stage treatment options are exceedingly limited and ineffective at prolonging life. We can now provide patients with a much-needed treatment option," he said.
David Hall, an MDS patient and chairman of the MDS UK Patient Support Group, said his organisation was "delighted" with the approval. "Each year in Scotland, approximately 200 people are diagnosed with MDS and this positive SMC decision will have a profoundly positive effect on their lives," he said.
- In February this year, the National Institute for Health and Clinical Excellence (NICE) overturned its previous decision not to recommend the use of Vidaza on the NHS in England and Wales after Celgene reduced the drug's price and resubmitted its proposed PAS for the drug.
Vidaza is indicated for the treatment of adult patients with the following conditions who are not eligible for hematopoietic stem cell transplantation: - intermediate-2 and high-risk MDS, according to the International Prognostic Scoring System; - chronic myelomonocytic leukaemia with 10% to 29% marrowblasts and without myeloproliferative disorder; and - acute myeloid leukaemia with 20% to 30% blasts and multi-lineage dysplasia, according to World Health Organisation (WHO) classification.