The National Institute for Health and Care Excellence (NICE) has issued new draft guidance not recommending Celgene's Revlimid (lenalidomide) for the treatment of myelodysplastic syndromes (MDS), but has also opened a public consultation on this preliminary decision.

MDS, which are diagnosed in around 2,000 people each year in England, are a group of bone marrow disorders characterised by the underproduction of one or more types of blood cells due to dysfunction of the marrow. They can lead to life-threatening diseases including acute myelogenous leukaemia (AML), as well as anaemia and increased risk of bleeding and infections.

This appraisal by NICE focuses on the use of Revlimid in the treatment of people with a specific type of MDS that is characterised by a chromosomal abnormality called a deletion 5q cytogenetic abnormality.

Celgene's Vidaza (azacitidine) was the first drug developed specifically for treating MDS and NICE points out that it recommends its use on the NHS. However, commenting on the draft guidance, NICE chief executive Sir Andrew Dillon added  that, for patients with this particular kind of MDS, the main treatment option at the moment is best supportive care, which includes blood transfusions.

NICE's independent appraisal committee heard from clinical experts that lenalidomide is an effective targeted therapy, with a real impact on the decreased need for blood transfusions, and the drug comes in tablet form, meaning that patients spend less time in hospital, said Sir Andrew.

However, he added: "data provided by the manufacturer showed uncertainty about whether lenalidomide actually extended these patients' lives. NHS resources are limited and NICE has to decide what treatments represent best value to the patient as well as the NHS, and in this case the manufacturer did not provide enough certain evidence to justify the price they are asking the NHS to pay."

NICE's appraisal committee agreed that the most plausible incremental cost-effectiveness ratio (ICER) for lenalidomide compared with best supportive care was likely to be greater than £70,000 per quality-adjusted life year (QALY) gained. The drug is available in 21-day packs of 10mg and 5mg capsules at net prices of £3,780 and £3,570 respectively, excluding VAT. The manufacturer estimated the cost of a 28-day cycle of treatment with 10mg of the drug (excluding VAT) to be £3,780.

The drug did not meet the criteria to be considered under end-of-life care, says NICE.

However, NICE has also announced that consultees, including the manufacturer, healthcare professionals and members of the public will now be able to comment on its preliminary recommendations, which are available for public consultation.

- Revlimid is Celgene's biggest earner; the firm's results for first-quarter 2013 show that the drug's sales in all markets rose 16% to just over $1 billion during the period, while Vidaza was up 10% to $204 million.