Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts

Azacitidine (Vidaza, Celgene) has a marketing authorisation for 'the treatment of adult patients aged 65 years or older who are not eligible for haematopoietic stem cell transplant with acute myeloid leukaemia with more than 30% marrow blasts, according to the World Health Organisation (WHO) classification'. Azacitidine also has a marketing authorisation for 'the treatment of adult patients who are not eligible for haematopoietic stem cell transplantation with acute myeloid leukaemia with 20% to 30% blasts and multi-lineage dysplasia, according to WHO classification', which is outside the scope of this appraisal.

The summary of product characteristics notes that azacitidine is most commonly associated with haematological reactions (71.4%) including thrombocytopenia, neutropenia and leukopenia (usually grade 3 to 4) and gastrointestinal events (60.6%) including nausea, vomiting (usually grade 1 to 2) or injection site reactions. For full details of adverse reactions and contraindications, see the summary of product characteristics.

Azacitidine is available at a list price of £321.00 per 100‑mg vial (excluding VAT, BNF, March 2016). It is administered subcutaneously at a recommended dose of 75 mg/m² body surface area per day for 7 days, followed by a rest period of 21 days (28‑day treatment cycles). The summary of product characteristics recommends that patients should have a minimum of 6 cycles. The drug cost of a cycle of azacitidine treatment is £4,494.00 (excluding VAT) assuming the list price, 7 treatments in a cycle, vial wastage and a body surface area of 1.8 m².

The company has agreed a patient access scheme with the Department of Health. This scheme provides a simple discount to the list price of azacitidine with the discount applied at the point of purchase or invoice. The level of the discount is commercial in confidence. The Department of Health considered that this patient access scheme does not constitute an excessive administrative burden on the NHS.