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NICE appraisal of eltrombopag for the treatment of chronic immune (idiopathic) thrombocytopenic purpura

In draft guidance published by NICE today (14 September 2010), eltrombopag (Revolade, GlaxoSmithKline) is not recommended for treating chronic immune (idiopathic) thrombocytopenic purpura[1] (ITP) in splenectomised adults[2] who do not respond to other treatments (for example, corticosteroids[3], immunoglobulins[4]), or as a second-line treatment for non-splenectomised adults when surgery is not advised.

The independent Appraisal Committee concluded that although eltrombopag can raise platelet levels[5], the evidence regarding the long term effectiveness of eltrombopag for this chronic condition is unclear. The cost of eltrombopag in relation to the estimated health benefits was also far greater than what is normally considered a cost effective use of NHS resources. Eltrombopag provides health gains at a cost of £104,100 per quality adjusted life year (QALY) for splenectomised people, and £116,750 per QALY for non-splenectomised people.

Dr Carole Longson, Health Technology Evaluation Centre Director at NICE said: “The Appraisal Committee recognise that for people living with ITP, the risk of bleeding, and subsequent bruising, can have a significant, detrimental effect on daily life. Unfortunately, even though the committee reviewed updated evidence provided by the manufacturer following the consultation, the committee felt there was still not sufficient evidence to recommend eltrombopag as a treatment for ITP. From the clinical evidence presented, it is not clear how much long term health benefit eltrombopag provides, compared with current alternative treatments available. Given this and the high cost of eltrombopag in relation to the estimated health benefits the committee concluded that it could not recommend eltrombopag for people with chronic ITP.”

NICE has not yet issued final guidance to the NHS; these decisions may change after appeal.

Until NICE issues final guidance, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its guidance on a technology it replaces local recommendations across the country.

Final guidance is likely to be published in October 2010.

Ends.

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Notes to Editors

About the appraisal

The final appraisal determination (FAD) is available at http://guidance.nice.org.uk/TA/Wave17/14

  • Eltrombopag (Revolade, GlaxoSmithKline) increases platelet production through activation of the thrombopoietin receptor. By stimulating platelet production, it helps to reduce bleeding.
  • Eltrombopag has UK marketing authorisation for the treatment of adult chronic immune (idiopathic) thrombocytopenic purpura (ITP) in splenectomised patients who are not responding to other treatments (e.g. corticosteroids, immunoglobulins) and as a second line treatment for adult non-splenectomised patients where surgery is contraindicated.
  • Eltrombopag is for oral administration. The summary of product characteristics (SPC) states that the recommended initial dose is 50 mg once daily. If after 2-3 weeks of initial therapy, the platelet counts are below the clinically targeted levels (50 × 109 per litre), the dose may be increased to a maximum of 75 mg once daily. Treatment should be discontinued if the platelet count does not increase sufficiently to avoid clinically important bleeding after 4 weeks of therapy at 75 mg. For full details of dose and administration, see the SPC.
  • The price per 50 mg tablet of eltrombopag is £55.00. It is available in 28-tablet packs containing 25 mg tablets (£770.00) or 50 mg tablets (£1540.00). Therefore, the cost of a four week course of treatment is between £1540.00 and £2310.00, and the annual cost is between £22,020.00 and £33,030.00. Costs may vary in different settings because of negotiated procurement discounts.
  • About 24 per 100,000 adults have ITP. It is more common in women. Among both women and men, incidence is higher in older ages.
  • In adults, ITP comes on gradually and it usually does not follow a viral illness. There may be no symptoms, mild bruising or bleeding, or severe bleeding.
  • Because most adults with ITP do not have any symptoms, ITP is usually diagnosed on a routine blood test that has been done for other reasons. The full blood count shows a lower number of platelets than normal.

About NICE

  • The National Institute for Health and Care Excellence (NICE) is the independent organisation responsible for providing national guidance on the promotion of good health and the prevention and treatment of ill health.
  • NICE produces guidance in three areas of health:

1. Ppublic health - guidance on the promotion of good health and the prevention of ill health for those working in the NHS, local authorities and the wider public and voluntary sector.

2. Health technologies - guidance on the use of new and existing medicines, treatments and procedures within the NHS.

3. Clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

[1] Chronic immune (idiopathic) thrombocytopenic purpura is a bleeding disorder caused by abnormally low levels of platelets in the blood. The disorder has no apparent cause.

[2] Splenectomised adults have had their spleen surgically removed; the spleen is an organ that is part of the lymphatic system. In some cases of ITP, a splenectomysplenectom is recommended, which can increase the platelet count in patients with the disorder.

[3] Corticosteroids are a type of steroid, which in turn is a type of hormone. Corticosteroids are commonly used to reduce inflammation, suppress the immune system, and replace hormones in the body.

[4] Immunoglobulins are also know as antibodies and are used by the immune system to identify and neutralise bacteria and viruses.

[5] Platelets are needed for the blood to clot. Normal platelet levels are between 150 and 400 × 109 per litre of blood. Low platelet counts (below 30 × 109 per litre) can result in bleeding and bruising.

This page was last updated: 13 September 2010

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Copyright 2014 National Institute for Health and Care Excellence. All rights reserved.