NICE consults on preliminary recommendations for a new treatment for enlarged spleen in adults with myelofibrosis

NICE has today (13 February) issued new draft guidance not recommending ruxolitinib (Jakavi, Novartis), for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary myelofibrosis, or myelofibrosis secondary to polycythaemia vera or essential thrombocythaemia [i].

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[ii]. 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.

The independent 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.

The Committee concluded that although it was plausible that ruxolitinib could offer a survival benefit, it noted uncertainties in the data used by the manufacturer to estimate the degree of survival benefit.

The Committee also noted the Evidence Review Group's (ERG) concerns around the limitations of the manufacturer's economic model and many of the modelling assumptions. The Committee concluded that there were fundamental issues with the structure of the manufacturer's model, and that the associated assumptions increased the uncertainty of the incremental cost-effectiveness ratio (ICER) and that rectifying them would increase the ICER. The Committee considered that, on balance, the ICER was likely to be closer to £149,000 per quality-adjusted life year (QALY) gained, rather than £74,000 per QALY gained, as presented by the manufacturer.

The Committee concluded that ruxolitinib was clinically effective 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

Commenting on the draft guidance, Professor Carole Longson, Health Technology Evaluation Centre Director at NICE said: “Myelofibrosis and splenomegaly can be extremely debilitating, with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 preliminary recommendations, 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 draft guidance is now out for consultation and I would urge all those with an interest in myelofibrosis to comment via the NICE website.”

NICE has not yet issued final guidance to the NHS; these decisions may change after consultation. Consultees, including the manufacturer, healthcare professionals and members of the public are now able to comment on the preliminary recommendations which are available for public consultation. Comments received during this consultation will be fully considered by the Committee at its next meeting in March and, following this meeting, the next draft guidance will be issued.

This draft guidance does not mean that people currently taking ruxolitinib will stop receiving it. 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 June 2013.


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. The main types of MPD are: primary myelofibrosis, polycythaemia vera and essential thrombocythaemia.


About the guidance

  1. The draft guidance will be available from 00:01 hrs on Wednesday 13 February until Tuesday 5 March 2013. Final guidance is expected to be published in June 2013.
  2. 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. This corresponds to an annual cost of approximately £43,200 per patient.
  3. 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'.
  4. 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.
  5. The Committee noted that it may be plausible to apply end-of-life criteria to a subgroup of patients with myelofibrosis classified by IPSS as high risk. However, no analysis was provided for this subgroup and therefore the Committee agreed that end-of-life criteria did not apply.
  6. A patient access scheme was not submitted by the manufacturer.
  7. The SMC in Scotland has not yet appraised ruxolitinib.
  8. NICE technology appraisals apply in England and Wales and are usually disseminated in Northern Ireland after local review.

About NICE

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:

  • 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
  • social care - the Health and Social Care Act (2012) sets out a new responsibility for NICE to develop guidance and quality standards for social care. To reflect this new role, from 1 April 2013 NICE will be called the National Institute for Health and Care Excellence (NICE) and it will become a Non-Departmental Public Body.

3. NICE produces standards for patient care:

  • quality standards - these describe high-priority areas for quality improvement in a defined care or service area
  • 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
  • CCG Outcomes Indicator Set(formerly known as COF) - NICE develops the potential clinical health improvement indicators to ensure quality of care for patients and communities served by the clinical commissioning groups (CCGs).

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: 12 February 2013

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