NICE rejects Revlimid for myelodysplastic syndromes

Celgene’s Revlimid has been rejected by NICE for use in treating myelodysplastic syndromes (MDS), a group of bone marrow disorders.

NICE first rejected the drug for this indication in October last year, citing lack of cost effectiveness. Celgene responded with a discounted price Patient Access Scheme, but it took until April for the firm and NICE to agree on a cost effectiveness model.

After this protracted process, NICE has again rejected the drug, once more citing cost grounds, but also expressing doubts about its clinical benefits compared to standard treatment.

Myelodysplastic syndromes (MDS), are diagnosed in around 2000 people each year in England and are characterised by the underproduction of one or more types of blood cells due to dysfunction of the marrow. MDS can lead to life threatening disease including acute myeloid leukaemia (AML), as well as anaemia and increased risk of bleeding and infections.

This appraisal focused on the use of Revlimid (lenalidomide) for treating people with a specific type of MDS caused by a chromosomal abnormality called a deletion 5q cytogenetic abnormality. Patients with this sub-type of MDS who are have increased transfusion-dependence, are usually given best supportive care, which includes blood transfusions.

Sir Andrew Dillon, NICE Chief Executive, said: “The committee heard from clinical experts that lenalidomide is an effective therapy. However, the data provided by the company showed uncertainty about whether lenalidomide actually extended lives.”

Consultees, including the manufacturer, healthcare professionals and members of the public are now able to comment on the preliminary recommendations which are available for public consultation, the closing date for comments being 11 June 2014.

The Committee concluded that lenalidomide is a clinically effective treatment for patients with this sub-type of the disease, but said there remained uncertainty about the overall survival benefit of lenalidomide. Despite this, the Committee agreed a survival benefit was plausible.

The Committee noted that without the patient access scheme and without treatment interruptions the cost per QALY was £68,000 for lenalidomide compared with best supportive care. The Committee therefore concluded that if there were concerns in establishing when the patient access scheme would come into effect, the cost per QALY could be considerably higher than £25,300 per QALY gained as presented by the manufacturer, because of the uncertainty associated with patient access scheme.

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