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NICE Says Yes in Draft Guidance to Eltrombopag for the Treatment of Chronic Immune (idiopathic) Thrombocytopenic Purpura

In draft guidance published today (18 December) by NICE, eltrombopag (Revolade, GlaxoSmithKline) is recommended within its marketing authorisation for treating chronic immune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 immunoglobulinsiv) or
•as second-line treatment, in adults who have not had a splenectomy because surgery is contraindicated.

•if the manufacturer provides eltrombopag with the discount agreed in the patient access scheme.
NICE conducted a review of the original guidance, published in October 2010, because the manufacturer submitted a patient access scheme.

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.

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 NICE is, therefore, pleased to be able to recommend eltrombopag for this condition in draft guidance, which is now out for consultation.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

The draft guidance (appraisal consultation document / ACD) can be found from Tuesday 18 December on the NICE website.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 May 2013.


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)splenectom 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 18 December.

2. Closing date for comments is Tuesday 22 January 2013. The second appraisal committee meeting is Wednesday 20 February 2013

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

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

5. 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.

6. 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.

7. 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.

8. 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. The Department of Health considered that this patient access scheme does not constitute an excessive administrative burden on the NHS. The manufacturer has agreed that the patient access scheme will remain in place until any review of this NICE technology appraisal guidance is published.

9. 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.

10. 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

11. 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.

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

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

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

•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).
About NICE
15. The National Institute for Health and Clinical Excellence (NICE) is the independent organisation responsible for providing national guidance and standards on the promotion of good health and the prevention and treatment of ill health.

16. 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, medical technologies (including devices and diagnostics) and procedures within the NHS
•clinical practice - guidance on the appropriate treatment and care of people with specific diseases and conditions within the NHS.
17. NICE produces standards for patient care:

•quality standards - these reflect the very best in high quality patient care, to help healthcare practitioners and commissioners of care deliver excellent services
•Quality and Outcomes Framework - NICE develops the clinical and health improvement indicators in the QOF, the Department of Health scheme which rewards GPs for how well they care for patients.
18. NICE provides advice and support on putting NICE guidance and standards into practice through its implementation programme, and it collates and accredits high quality health guidance, research and information to help health professionals deliver the best patient care through NHS Evidence.


Posted: December 2012