The UK’s National Institute for Health and Clinical Excellence (NICE) has recommended against the use of azacitidine in England and Wales for certain haematological conditions, including strains of leukaemia, in draft guidance. Azacitidine is marketed by Celgene under the brand name Vidaza.
An advisory committee for the regulatory body said that the treatment should not be an option for adult patients affected by conditions outlined in the proposed market authorisation who are ineligible for haematopoietic stem cell transplantation.
Azacitidine is intended as a treatment for intermediate-2 and high-risk myelodysplastic syndromes (MDS) – a collection of haematological disorders which combine to cause ineffective production of myeloid blood cells – as well as certain variations of both chronic myelomonocytic leukaemia and acute myeloid leukaemia.
The new guidance follows an appeal from Celgene challenging a March 2010 decision from NICE to rule against recommending the drug.
As with the earlier ruling, the committee acknowledged that, on the basis of clinical evidence, azacitidine is an effective treatment for the conditions, and has several benefits when compared to other treatments.
However, the drug was not seen as a cost-effective treatment for MDS, chronic myelomonocytic leukaemia or acute myeloid leukaemia, even under a patient access scheme which would see Celgene offer the drug with a seven per cent reduction in the acquisition cost.
It is recommended patients undergo a minimum of six cycles of treatment, with each cycle costing £4,494, excluding administration costs.
A consultation process is now ongoing, with NICE inviting comments on the draft guidance up to November 25.
A second appraisal meeting will take place on January 6, 2011.
NICE has more information on how to submit comments, as well as detailed notes on the guidance.