Negative draft NICE guidance on Celgene’s Revlimid for myelodysplastic syndromes

The UK drug watchdog the National Institute for Health and Care Excellence (NICE)  today (October 24) issued final draft guidance that does not recommend US biotech firm Celgene’s (Nasdaq: CELG) blockbuster multiple myeloma drug Revlimid (lenalidomide) for treating some myelodysplastic syndromes (MDS).

Sales of Revlimid in its currently approved indications are expected to top $4.30 billion this year and were up 13% in the second quarter of 2013 to $1.05 billion ( The Pharma Letter July 25).

MDS are diagnosed in around 2,000 people each year in England. They are a group of bone marrow disorders characterized 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 leukemia (AML), as well as anemia and increased risk of bleeding and infections.

This appraisal focuses on the use of lenalidomide for treating people with a specific type of MDS that is characterized by a chromosomal abnormality called a deletion 5q cytogenetic abnormality.

Commenting on the draft guidance, Sir Andrew Dillon, the NICE’s chief executive, said: “Azacitidine was the first drug developed specifically for treating another type of MDS and it is recommended for use by the NHS [National Health Service] by NICE. At the moment, for patients with the particular kind of MDS considered in this appraisal, the main treatment option is best supportive care, which includes blood transfusions.”

Questions of actual extended lives

He continued: “The committee heard from clinical experts that lenalidomide is an effective targeted therapy. However, the data provided by the company showed uncertainty about whether lenalidomide actually extended lives. NHS resources are limited and NICE has to decide what treatments represent best benefit 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.”

Until final guidance is issued, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations across the country. This draft guidance does not mean that people currently taking lenalidomide will stop receiving it. They have the option to continue treatment until they and their clinicians consider it appropriate to stop.

The NICE 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 ($94,460) per Quality Adjusted Life Year (QALY) gained. Lenalidomide 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 lenalidomide (excluding VAT) to be £3,780.