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

In preliminary guidance published today (10June 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 second-line treatment for non-splenectomised adults when surgery is not advised.

The independent Appraisal Committee considered the use of eltrombopag in two groups of people - those who do and those who do not have persistent bleeding problems. The committee concluded that, although eltrombopag can raise platelet levels[5], the evidence was very unclear about how many health benefits eltrombopag would provide compared with other existing treatments. 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.

Gillian Leng, Deputy Chief Executive of NICE said: “The Appraisal Committee understood that for people living with ITP, the risk of bleeding can have a considerable effect on their daily activities and also prevent or delay surgery. But unfortunately, the committee felt there was not sufficient evidence to recommend eltrombopag as a treatment for ITP. There are existing treatments available for ITP and, from the clinical evidence, it is very unclear how much health benefit eltrombopag provides, compared with these current alternative treatments. The cost of eltrombopag in relation to the estimated health benefits is too high to be a cost-effective use of NHS resources. The committee therefore concluded that it could not recommend eltrombopag for people with chronic ITP.”

This draft guidance has been issued for consultation and interested parties now have an opportunity to comment on the draft recommendations made by the independent appraisal committee; NICE has not yet issued final guidance to the NHS.

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 xxxxxxxx 2010.


[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.

[3] Corticosteroids are a type of steroid, which are in turn 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.

Ends

Notes to Editors

About the appraisal

  • The appraisal consultation document (ACD) is available for comment from 5.00pm on Thursday 10 June to 5.00pm on Thursday 1 July at 5.00pm at http://guidance.nice.org.uk/TA/Wave17/14/Consultation/Latest.
  • After consultation the Appraisal Committee will meet again on Tuesday 27 July to consider the evidence, this appraisal consultation document and any comments from the consultees.
  • 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 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. 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 draft summary of product characteristics (SPC).
  • The manufacturer's submission states that the price per 50 mg tablet is £55.00. Eltrombopag 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.
  • In adults, ITP occurs in about 6 in 100,000 people. It is more common in women and predominantly occurs in those of child bearing age. It may also be seen more often in the elderly.
  • 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:
    • Public 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.
    • Health technologies - guidance on the use of new and existing medicines, treatments and procedures within the NHS.
    • Clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.

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This page was last updated: 10 June 2010

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

Accessibility | Cymraeg | Freedom of information | Vision Impaired | Contact Us | Glossary | Data protection | Copyright | Disclaimer | Terms and conditions

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