MPD Voice
Ruxolitinib as a treatment for Myelofibrosis (MF) – NICE draft guidance
MPD Voice has been active in supporting trials using Ruxolitinib for the treatment of MF and have reported on the trials at forums.  We would like to bring to your attention the draft guidance issued by NICE (National Institute for Health and Clinical Excellence) regarding Ruxolitinib. 

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NICE has today (13 February) issued new draft guidance not recommending ruxolitinib (Jakavi, Novartis), for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary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.

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. 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.

Professor Claire Harrison, Guy’s & St Thomas’ Hospital, has issued the following quote in reply to this draft guidance.
“The lives of patients affected by myelofibrosis are improved with ruxolitinib therapy.  In many cases this improvement is dramatic with long lasting tangible benefits.  There is now increasing evidence that ruxolitinib therapy also prolongs survival in this difficult disease where we have previously had very limited options", said Professor Claire Harrison, consultant haematologist and co-founder of patient charity, MPD Voice.  “This decision is disappointing for patients, their families and clinicians.  However the work of NICE is crucial and we will work with NICE as well as patients and clinicians to ensure that as drugs are developed that appropriate information is gathered so that important new groundbreaking treatments like ruxolitinib are made available where appropriate to patients in the UK.”
If you have any questions regarding this draft guidance please contact us at
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