NICE has today (21 March) published draft guidance on eltrombopag (Revolade, GlaxoSmithKline).

Eltrombopag is licensed for treating chronic immune (idiopathic) thrombocytopenic purpuraⁱ (ITP) in adults who have had a splenectomyⁱⁱ, and whose condition is refractory to other treatments (for example, corticosteroidsⁱⁱⁱ or immunoglobulins^iv), and as a second-line treatment in adults who have not had a splenectomy because surgery is contraindicated.

In the draft guidance, NICE has recommended eltrombopag as an option for both of these patient groups, only if:

• their condition is refractory to standard active treatments and rescue therapies, or
• they have severe disease and a high risk of bleeding that needs frequent courses of rescue therapies.

The treatment is recommended for NHS use only if the manufacturer makes it available to the NHS under the terms agreed with the Department of Health as part of a patient access scheme.

NICE conducted a review of the original guidance, published in October 2010, because the manufacturer submitted a patient access scheme. NICE is today issuing a second appraisal consultation document because the independent Appraisal Committee needs to consult on comments received on the first ACD and its response to those comments.

The Committee concluded that there are few treatment options licensed for people with chronic ITP and that eltrombopag, along with romiplostim (which NICE recommended in 2011), represents an effective approach to treating the condition.

Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: “Living with ITP can have a significant effect on daily life and those affected are at risk of bleeding and subsequent bruising. NICE is, therefore, pleased to be able to recommend eltrombopag for this condition in draft guidance, which is now out for consultation. The next step is for stakeholders to submit their comments on it via the NICE website.”

NICE has not yet issued final guidance to the NHS; these decisions may change after consultation. 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 expected to be published in July 2013.

Ends

Notes to Editors

References and explanation of terms

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

ii. In some cases of ITP, removing the spleen, an organ that is part of the lymphatic system (the operation is called a splenectomy) is recommended. This can increase the platelet count in patients with the disorder.

iii. 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.

iv. Immunoglobulins are also known as antibodies and are used by the immune system to identify and neutralise bacteria and viruses. They also reduce platelet destruction; therefore injecting them into the bloodstream is used as a treatment for ITP.

v. 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.

About the draft guidance

1. The draft guidance is available from 00:01hrs on Thursday 21 March. Embargoed copies are available on request from the press office.

2. Closing date for comments is Monday 15 April 2013. The next appraisal committee meeting is Tuesday 23 April 2013.

3. Final guidance is expected to be published in July 2013.

4. In accordance with the guide to the single technology appraisal process (section 3.5.34), NICE had to extend the timelines for this appraisal to allow for a full and comprehensive review of new comments received in response to the first ACD.

5. The first ACD on eltrombopag for the treatment of chronic immune (idiopathic) thrombocytopenic purpura issued for consultation in December 2012 can be found at on the NICE website.

6. Eltrombopag (Revolade, GlaxoSmithKline) increases platelet production through activation of the thrombopoietin receptor. By stimulating platelet production, it helps to reduce bleeding.

7. 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 non-splenectomised patients where surgery is contraindicated.

8. Eltrombopag is taken orally. The summary of product characteristics (SPC) states that the recommended initial dose is 50 mg once daily (patients of East Asian ancestry should start eltrombopag at a reduced dose of 25 mg once daily). Patients should take eltrombopag at least 4 hours before or after any products such as antacids, dairy products (or other calcium-containing food products) or mineral supplements containing polyvalent cations (for example, iron, calcium, magnesium, aluminium, selenium and zinc). If, after initial therapy, platelet counts are below the clinically targeted level (50×109 per litre), the dosage may be increased to a maximum of 75 mg once daily. Treatment should be stopped if the platelet count does not increase sufficiently to avoid clinically significant bleeding after 4 weeks of therapy at a dosage of 75 mg once daily. For full details of dosage and administration, see the summary of product characteristics.

9. The ‘British National Formulary' (BNF; edition 64) states that the net price of a 28-tablet pack of 25 mg eltrombopag is £770 (a single 25 mg dose costs £27.50). The net price of a 28-tablet pack of 50 mg eltrombopag is £1540 (a single 50 mg dose costs £55). The cost per patient will vary with dose adjustment and treatment duration. The manufacturer indicated that the average daily cost of eltrombopag (based on the mean dose of eltrombopag in the EXTEND study of 51.3 mg per day) is £56.43.

10. The manufacturer of eltrombopag (GlaxoSmithKline) has agreed a patient access scheme with the Department of Health that makes eltrombopag available with a discount. The size of the discount is commercial in confidence at the request of the manufacturer. The manufacturer has agreed that the patient access scheme will remain in place until any review of this NICE technology appraisal guidance is published.

11. The UK incidence of adult ITP is estimated to be around 120 per year and 3000-3500 people are affected at any one time England and Wales. It is more common in women. Among both women and men, incidence is higher in older ages.

12. 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. ITP is not normally fatal but some rare cases may be life-threatening because of a risk of spontaneous haemorrhages.

13. 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.

14. NICE published guidance (TA237) in 2010 not recommending eltrombopag for the treatment of chronic immune or idiopathic thrombocytopenic purpura.

15. Further information on the review process is given in the NICE Single Technology Appraisal process guide, section 6.

16. Until final guidance is published, the current guidance on eltrombopag, TA205, is applicable.

Related NICE guidance

• Romiplostim for the treatment of chronic immune or idiopathic thrombocytopenic purpura. NICE technology appraisal guidance 221 (2011).
• Eltrombopag for the treatment of chronic immune or idiopathic thrombocytopenic purpura. NICE technology appraisal guidance 205 (2010).

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