Celgene has announced that the National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination (FAD) on its oral blood cancer treatment, lenalidomide (Revlimid®) for transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate. 

The decision marks the end of a lengthy appraisal process, which has lasted more than 18 months, but means that patients in England and Wales will have full access in the long term to the only medicine specifically licensed for this rare but debilitating disease.

Commenting on the FAD, Dr Adrian Kilcoyne, Medical Director, Celgene UK & Ireland said: “We are extremely pleased with this recommendation from NICE. Up until now, it has only been patients in England who could access lenalidomide via the Cancer Drugs Fund and not patients in Wales. Today’s decision, therefore, hopefully eases pressure on the Cancer Drugs Fund. It recognises the value of medical research and innovation, a great contributor to increased life expectancy. It also means that NICE and the Scottish Medicines Consortium (SMC) are now aligned since the SMC issued advice in March this year, which has helped patients in Northern Ireland, as well as Scotland, obtain the treatment.”

MDS are a heterogeneous group of cancers where the production of blood cells by the bone marrow is disrupted, often leading to infections, bleeding and severe anaemia, which can cause severe fatigue.¹

In general, MDS del(5q) is associated with a poor prognosis – especially when other cytogenetic abnormalities are present – including the risk of progressing to acute myeloid leukaemia (AML), which is often fatal.

Red blood cell transfusions are the mainstay of disease management as there are no other medicines licensed for the treatment of low-to-intermediate-risk MDS.

Around 40% of patients are red blood cell transfusion dependent when they are diagnosed and the majority of patients become transfusion dependent at some stage during their disease.²

This imposes significant restrictions on the lives of patients and negatively impacts on their overall Quality of Life.³

However, treatment with lenalidomide has been shown to help some patients manage their condition without blood transfusions for periods of time.⁴

Consequently, today’s decision from NICE for lenalidomide is important as blood transfusions can place a very real burden on patients and the National Health Service.

The final draft guidance was based on the results of MDS-004, the largest global trial of MDS del(5q) patients conducted to date.

This showed that a significantly larger proportion of patients achieved the primary endpoint of transfusion independence (>6 months) on lenalidomide 10mg given on 21 days of 28 day cycles compared with placebo (57.4% vs. 2.2%).⁵

In addition, even though overall survival was not the primary endpoint of the MDS-004 trial and patients on the placebo arm crossed over to be treated with lenalidomide early on in the study, patients who achieved transfusion independence of 2 months or more had a greater than 40% reduction in the relative risk of death.

Celgene received ministerial approval on a patient access scheme to improve the cost-effectiveness of lenalidomide in MDS isolated del(5q) in June 2013.

This involves the NHS paying for lenalidomide for up to 26 monthly cycles. 

Celgene will then provide the treatment free of charge for those people who receive more than 26 monthly cycles.

Following approval from the NICE Executive, the FAD should now form the NICE guidance for lenalidomide in MDS del(5q) in September 2014.

References

1. Kurzrock R. Semin Hematol 2002; 39(Suppl 2): 18-25​
2. Brechignac S et al. Blood 2004; 104 (11): 4716
3. Valent P et al. European Journal of Clinical Investigation 2008; 38(3): 143–149
4. Fenaux P, et al. Blood 2011; 118(14): 3765-76
5. Giagounidis A, et al. European Journal of Haematology 2014; Published Online First, 9 June 2014
6. Revlimid Summary of Product Characteristics. Available here. [Last accessed August 2014]

Source: Calgene