The MDS Beacon™

The United Kingdom’s National Institute of Clinical Excellence (NICE) released Monday that it will not be recommending state funding for Vidaza (azacitidine). Patients in England and Wales with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia, and acute myeloid leukemia, the three conditions for which the drug is approved, will not be able to receive the drug unless the manufacturer reduces the cost of treatment.

The European Medicines Agency (EMEA) approved Vidaza for European markets in March of this year. For use of Vidaza in the U.K., the drug manufacturer, Celgene, is required to compile a report on the drug’s clinical results and an estimate for the cost of treatment.

Celgene’s report was based on results from a successful Phase 3 clinical trial, in which patients taking Vidaza experienced a 9.6 month extension in overall survival compared to conventional care.

The NICE Appraisal Committee is a group of doctors, professionals, and patient experts who review the report and make a recommendation. After consultation and review, the Committee decided that the cost of treatment prevents the drug from being an effective use of National Health Service (NHS) resources.

The Committee agreed that Vidaza’s clinical results show it is effective in extending patient lives. They also acknowledged that the drug extends the average time until MDS transforms to acute myeloid leukemia and that it decreases the number of patients dependent on blood transfusions. The Committee concluded that Vidaza is a clinically effective treatment for MDS.

However, the Committee concluded that Vidaza will likely cost significantly more than the amount Celgene estimated it will cost the NHS to provide the drug to patients. The Committee estimates the drug will not be cost-effective. Therefore, they currently cannot recommend state funding for Vidaza.

NICE’s guidance is preliminary. The Committee will have a second meeting to review the drug on September 3. In this meeting, they will consider the clinical evidence, their preliminary recommendations, and comments by people outside of the Committee. Comments are accepted from patients, health care professionals, and the general public and can be made to the Committee until August 24.

After the second meeting, they will prepare the final recommendation for the NHS. However, NICE does not anticipate a large enough decrease in cost estimates to change the recommendation.

David Hall, the Chairman of the MDS U.K. Patient Support Group, released a statement to PharmaTimes saying that patients in the U.K. will be “devastated” by the recommendation. He added that when patients are suffering in old age and facing a premature death, “Our society needs to look critically at the avenues through which public funds are dispensed.”

To view details on the Committee’s conclusions and information about the appraisal process or to submit a comment, visit the Committee’s recommendation on the NICE Web site.