NICE proposes to recommend lenalidomide for myelodysplastic syndromes

NICE proposes to recommend lenalidomide for myelodysplastic syndromes

NICE has issued final draft guidance recommending lenalidomide (also known as Revlimid and marketed by Celgene) as an option, for treating myelodysplastic syndromes.

Myelodysplastic syndromes (MDS), which are diagnosed in around 2000 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. 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 lenalidomide for treating people with a specific type of MDS that is characterised by a chromosomal abnormality called an isolated deletion 5q cytogenetic abnormality. At the moment the main treatment option for people with the particular kind of MDS considered in this appraisal is best supportive care including regular blood transfusions.

Commenting on the final draft guidance, Sir Andrew Dillon, NICE chief executive, said: "The committee heard from clinical experts that lenalidomide is an effective therapy. Celgene – who market lenalidomide – worked with us to provide enough evidence to make it possible for us to recommend it for this group of people.

"Celgene provided a revised analyses and further information on their proposal for a reduction in the cost of the drug to the NHS (patient access scheme)."

Celgene's patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. The company will provide the drug free of charge for those people who receive more than 26 monthly cycles.

The draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments.

Ends

Notes to Editors

Explanation of terms

1.    Lenalidomide holds a marketing authorisation for treating transfusion-dependent anaemia caused by low or intermediate-1 risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.

About the guidance

NICE technology appraisal 218 guidance recommends azacitadine as a treatment option for patients with intermediate-2 or high risk MDS who are not eligible for haematopoietic stem cell transplantation, which is a different population to the one considered in this appraisal.
The main treatment option currently available for people with low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate is best supportive care, which involves regular red blood cell transfusions.
The Committee concluded that lenalidomide is a clinically effective treatment for people with low- or intermediate-1-risk MDS associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate, because it was associated with a statistically significant improvement in transfusion independence and health related quality of life compared with placebo. The Committee agreed that it was plausible for lenalidomide to indirectly improve overall survival by improving transfusion independence.
The Committee noted that the revised company's base-case incremental cost-effectiveness ratio (ICER) for lenalidomide compared with best supportive care with the patient access scheme was £25,300 per QALY gained. The Committee noted that the patient access scheme was not a simple discount and that would only benefit those on treatment after 26 cycles. The Committee agreed that because the proportion of people on treatment beyond 26 cycles was uncertain so were the potential cost savings from the patient access scheme, and noted that if the proportion of people who reached 26 cycles was less than 27%, the ICER would be greater than £30,000 per QALY gained.
The Committee agreed that the ICER was uncertain, because of the patient access scheme, but accepted that a commitment from the company to publish data on the proportion of people on treatment beyond 26 cycles would provide reassurance that lenalidomide for treating MDS associated with an isolated deletion 5q cytogenetic abnormality was recommended as a cost-effective use of NHS resources.

Lenalidomide is available in 21-day packs of 10 mg and 5 mg capsules at net prices of £3780 and £3570 respectively. The cost of a 28-day cycle of treatment with 10 mg of lenalidomide (excluding VAT) is £3,780. Celgene has agreed a standard patient access scheme with the Department of Health, in which the NHS pays for lenalidomide treatment for up to 26 monthly cycles. The company subsequently provides free of charge lenalidomide for those people who receive more than 26 monthly cycles.
About NICE

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for driving improvement and excellence in the health and social care system. We develop guidance, standards and information on high-quality health and social care. We also advise on ways to promote healthy living and prevent ill health.

Our aim is to help practitioners deliver the best possible care and give people the most effective treatments, which are based on the most up-to-date evidence and provide value for money, in order to reduce inequalities and variation.

Our products and resources are produced for the NHS, local authorities, care providers, charities, and anyone who has a responsibility for commissioning or providing healthcare, public health or social care services.

To find out more about what we do, visit our website: www.nice.org.uk and follow us on Twitter: @NICEComms.

About NICE

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for driving improvement and excellence in the health and social care system. We develop guidance, standards and information on high-quality health and social care. We also advise on ways to promote healthy living and prevent ill health.

Our aim is to help practitioners deliver the best possible care and give people the most effective treatments, which are based on the most up-to-date evidence and provide value for money, in order to reduce inequalities and variation.

Our products and resources are produced for the NHS, local authorities, care providers, charities, and anyone who has a responsibility for commissioning or providing healthcare, public health or social care services.

To find out more about what we do, visit our website:www.nice.org.uk and follow us on Twitter: @NICEComms.

The committee heard from clinical experts that lenalidomide is an effective therapy.
Sir Andrew Dillon, NICE chief executive
Related Resources

Myelodysplastic syndrome (deletion 5q) - lenalidomide

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