NICE gives green light to romiplostim for the treatment of chronic immune (idiopathic) thrombocytopenic purpura (ITP)
The National Institute for Health and Care Excellence (NICE) has recommended romiplostim (Nplate, Amgen) for the treatment of chronic immune (idiopathic) thrombocytopenic purpura (ITP) in some patients in draft guidance issued today (Thursday 17 March 2011).
ITP is a bleeding disorder in which the immune system destroys platelets, which are needed for normal blood clotting. People with the disease have abnormally low levels of platelets in the blood. The condition is currently thought to affect 3000 - 3500 people in the UK; some patients may only have the condition for a short period of time, but when a patient has ITP for over 12 months this is defined as having chronic ITP.
In this draft guidance, romiplostim is recommended for the treatment of adults with chronic ITP:
- whose condition does not respond to standard active treatments and rescue therapies or,
- who have severe disease, and a high risk of bleeding that requires frequent courses of rescue therapies, and
- if the manufacturer makes romiplostim available with the rebate on the list price agreed under the patient access scheme.
Only a haematologist should initiate and supervise treatment with romiplostim. The recommendations are conditional on the manufacturer providing romiplostim at a discounted cost as part of a patient access scheme.
NICE has not yet issued final guidance to the NHS; consultees now have the opportunity to appeal against this final draft guidance.
Dr Carole Longson, Director, Health Technology Evaluation Centre at NICE said: “ITP is a serious, sometimes debilitating disorder, and some of the current treatments have considerable side effects, so we are pleased to recommend the use of romiplostim as a clinically and cost effective treatment for some people with severe, chronic ITP in our draft guidance issued today. The manufacturer has also submitted a patient access scheme where the cost of the treatment will be reduced.”
The draft guidance is available from Thursday 17 March until Thursday 31 March.
Consultees now have the opportunity to appeal against this draft guidance. If there are no appeals, or an appeal is not upheld, the recommendations in the draft will be issued as final NICE guidance. NICE expects to issue final guidance to the NHS in April 2011. Until this time, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations across the country.
Final guidance is likely to be published in April 2011.
 Of unknown cause.
 Patient access schemes are ways pharmaceutical companies can propose to enable patients to gain access to high cost drugs.
Notes to Editors
About the guidance
1. The draft guidance is available from Thursday 17 March until Thursday 31 March
2. Romiplostim (Nplate, Amgen) is a protein that mimics the action of thrombopoietin (a glycoprotein hormone produced mainly by the liver and the kidneys that regulates the production of platelets by the bone marrow) by acting as an agonist at thrombopoietin receptors. It stimulates the differentiation and proliferation of bone marrow cells responsible for producing platelets (megakaryocytes), and so increases platelet production and platelet counts.
3. The summary of product characteristics (SPC) states that the recommended initial dose of romiplostim is 1 microgram/kg of actual body weight, administered once weekly as a subcutaneous injection. The dose may be adjusted by increments of 1 microgram/kg until a platelet count equal to or above 50 × 109 platelets per litre of blood is reached. A maximum dose of 10 micrograms/kg once weekly should not be exceeded. Platelet counts should be measured weekly until a stable count equal to or above 50 × 109 platelets per litre for at least 4 weeks without adjusting the dose. Thereafter, platelet counts should be measured monthly. Treatment with romiplostim should be stopped if the platelet count does not increase sufficiently to avoid clinically significant bleeding after 4 weeks of romiplostim therapy at the highest weekly dose of 10 micrograms/kg. Romiplostim should also be stopped if a peripheral blood smear indicates increased bone marrow reticulin (collagen-like protein fibres found in tissue) as well as if a loss of efficacy is observed.
4. Romiplostim costs £1.93 per microgram; therefore a 250 microgram vial costs £482 (excluding VAT; British national formulary [BNF] edition 60). The cost of treatment varies depending on the patient's weight, the dosing regimen and any waste that results from discarding any unused drug from the single use of a 250 microgram vial. The annual cost of romiplostim treatment for a person weighing 80 kg would be £8020 at a dose of 1 microgram/kg weekly and £80,204 at a dose of 10 micrograms/kg weekly (assuming no waste). The manufacturer of romiplostim (Amgen) has agreed a patient access scheme with the Department of Health which offers a rebate on the list price of the 250 microgram vial of romiplostim. The size of the rebate is commercial in confidence. The manufacturer has agreed that the patient access scheme will remain in place until any review of this NICE technology appraisal guidance is published.
5. The condition is currently thought to affect 3000 - 3500 people in the UK; some patients may only have the condition for a short period of time, but when a patient has ITP for over 12 months this is defined as having chronic ITP.
6. It is more common in women. Among both women and men, incidence is higher in older people.
7. 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.
8. 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.
9. There is further information on patient access schemes on the NICE website
1. The National Institute for Health and Care 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.
2. NICE produces guidance in three areas of health:
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- 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.
4. NICE provides advice and support on putting NICE guidance and standards into practice throughits 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.
This page was last updated: 16 March 2011