Success for rare cancer drug

04/08/10

The Appeal Panel of the National Institute for Health and Clinical Excellence (NICE) has allowed an appeal against a decision refusing to recommend reimbursement of a novel anti-cancer drug, azacitidine. Azacitidine is the only life-extending treatment available for many sufferers of a very rare group of leukaemias. NICE accepted that azacitidine increased life-expectancy by a median of 9 ½ months of high quality extra life.

The NICE Appraisal Committee had refused to recommend treatment with azacitidine as cost effective. However, the Appeal Panel accepted the submission of Celgene, the manufacturer, that omitting chemotherapy as a comparator treatment was perverse, since it is widely used in clinical practice. The NICE Appeal Panel only rarely allows such appeals. Two separate grounds of challenge alleging procedural unfairness and breach of the Human Rights Act were dismissed. Appeals were also brought by a patient group and by representatives of the Royal Colleges of Pathologists and of Physicians.

The Appraisal Committee must now reconsider its guidance, taking account of chemotherapy as a comparator.