The cost regulator for the NHS in England and Wales has said that Celgene’s Revlimid should be an option for treating patients with a particular form of myelodysplastic syndromes, a group of bone marrow disorders.

In new final draft guidance, the National Institute for Health and Care Excellence (NICE) has recommended use of Revlimid (lenalidomide) in a specific type of MDS that is characterised by a chromosomal abnormality called an isolated deletion 5q cytogenetic abnormality. Currently, the main treatment option for people with this kind of MDS is best supportive care including regular blood transfusions.

In earlier draft guidances published this May and in June last year, NICE had not backed Revlimid in this use because, while the Institute’s appraisal committee had found it to be an effective therapy, the data provided by Celgene showed uncertainty about whether it actually extended lives. The June 2013 draft guidance had also opened a consultation.

Commenting on the final draft guidance, NICE chief executive Sir Andrew Dillon said the committee had heard from clinical experts that the drug is an effective therapy, and that Celgene had worked with NICE to provide enough evidence to make it possible to recommend it for this group of people.

Celgene had provided NICE with a revised analysis and further information on its proposal for a reduction in the cost of the drug to the NHS, though a patient access scheme (PAS).

This PAS involves the NHS paying for treatment with the drug for up to 26 monthly cycles. The company will then provide it free for those people who receive more than 26 monthly cycles.

The Committee noted that the PAS was not a simple discount and would only benefit people who were on treatment after 26 cycles. As it was uncertain what proportion of those treated this group would represent, so were the potential savings of the PAS. However, the panel had accepted that a commitment from Celgene to publish data on the proportion of people on treatment beyond 26 cycles would provide reassurance that the drug’s use in treating MDS associated with an isolated deletion 5q cytogenetic abnormality was recommended as a cost-effective use of NHS resources.

- Around 2,000 people in England are diagnosed each year with MDS, which are characterised by the underproduction of one or more types of blood cells due to dysfunction of the bone marrow. MDS can lead to life-threatening diseases including acute myeloid leukemia, as well as anemia and increased risk of bleeding and infections.