NICE has published final guidance not recommending ruxolitinib (Jakavi, Novartis), for the treatment of disease-related splenomegaly (enlarged spleen) or its symptoms in adults with primary myelofibrosis, or post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis [i].

Myelofibrosis is a rare blood cancer in which the bone marrow produces too many cells too rapidly. The bone marrow becomes scarred making it less able to make blood cells. To compensate for this, other organs in the body, including the liver and the spleen, begin to produce them. As the spleen begins to produce blood cells, it becomes enlarged.

The independent Committee, a group of experts which developed the recommendations for NICE, concluded that ruxolitinib was clinically effective in reducing spleen size and symptoms such as itch and fatigue, but could not be considered a cost-effective use of NHS resources compared with best available therapy for treating disease-related splenomegaly or symptoms in adults with myelofibrosis. It concluded that although it was plausible that ruxolitinib could offer an overall survival benefit, the reason for this remained unclear because of uncertainties in the evidence.

Commenting on the guidance, Sir Andrew Dillon, Chief Executive at NICE said: “NICE is aware that myelofibrosis and splenomegaly can be extremely debilitating, with symptoms such as severe itching and fatigue. It is disappointing not to be able to recommend this new treatment in our final guidance, but in order to do this we have to be sure that the treatment is both clinically and cost effective, because money has to be diverted from elsewhere in the health service to pay for it.”

The Committee noted the Evidence Review Group's (ERG)[ii] concerns around the limitations of the manufacturer's economic model and many of the modelling assumptions. The Committee identified a number of important issues with the structure of the manufacturer's model, and felt the assumptions [iii] made in the model increased the uncertainty about the cost effectiveness of ruxolitinib.

The final guidance is available from Wednesday 26 June.

This is NICE's final guidance on this technology and now replaces local recommendations across the country.

Ends

Notes to Editors

References and explanation of terms

i. Myelofibrosis is a type of myeloproliferative disease (MPD). This is a disease in which the bone marrow produces too many cells too rapidly. Types of MPD include: primary myelofibrosis, polycythaemia vera and post essential thrombocythaemia. Myelofibrosis can develop from polycythaemia vera and post essential thrombocythaemia.

Myelofibrosis is a type of haematological (blood) cancer. It is a rare condition, with around 0.4 cases per 100,000 each year in the UK (patient.co.uk). The bone marrow becomes scarred (fibrosed) making it is less able to make blood cells. To compensate for this, other organs in the body, including the liver and the spleen, begin to produce them. As the spleen begins to produce blood cells, it grows in size causing splenomegaly (enlarged spleen).

ii. The Evidence Review Group (ERG) assess the strength and quality of the research evidence submitted by manufacturers to NICE as part of the technology appraisals process.

iii. The assumptions presented in the evidence included maintenance of response, survival and discontinuation rates over time, no disease progression, no complications for patients in the responder health state, and continuation of best available therapy throughout the time horizon.

About the guidance

1. The final guidance will be available from 00:01hrs on Wednesday 26 June.
2. The Committee considered that the ICER could be approaching £149,000 per quality-adjusted life year (QALY) gained as presented by the ERG, but acknowledged that this may have overestimated the ICER because of uncertainty in the evidence submitted by the manufacturer. It considered the base-case ICERs presented by the manufacturer (£74,000 and £57,000 per QALY gained) were likely to have underestimated the ICER because of the limitations of the evidence.
3. Ruxolitinib costs £3,600 for a 60-tablet pack of 15 mg or 20 mg tablets, or £1800 for a 60-tablet pack of 5 mg (excluding VAT; British National Formulary [BNF] online, November 2012). This corresponds to an annual cost of approximately £43,200 per patient.
4. Ruxolitinib (Jakavi, Novartis) has a UK marketing authorisation for ‘the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis'.
5. Ruxolitinib was considered by the Committee to be an innovative treatment for patients with splenomegaly and myelofibrosis. The technology is a targeted treatment and offers a step change in treating splenomegaly because it manages symptoms for which there is currently no available treatment.
6. The Committee considered that myelofibrosis symptoms (especially itch and fatigue) and spleen size are significant, and that improving these would be beneficial to the wellbeing of patients. The Committee concluded that ruxolitinib was effective in reducing spleen size and in treating symptoms, such as itch and fatigue.
7. The Committee concluded that it had not been provided with evidence that high-risk patients met the end-of-life criteria.
8. A patient access scheme was not submitted by the manufacturer.
9. Ruxolitinib (Jakavi®) is not recommended for use within NHS Scotland for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as chronic idiopathic myelofibrosis), post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis.
10. NICE technology appraisals apply across the NHS in England and Wales.

About NICE

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