MPN Voice
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Ruxolitinib as a treatment for Myelofibrosis (MF) – NICE draft guidance - UPDATE

Further to the NICE draft guidance not recommending Ruxolitinib (Jakavi, Novartis), for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary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.

MPD Voice has written to NICE regarding this decision, urging them most strongly to reconsider their decision as we feel this is a very important issue for all of us that have a MPD, especially as the latest results of the trials currently being undertaken are very positive.

The NICE Appraisal Committee has considered the evidence submitted by the manufacturer and the views of non-manufacturer consultees and commentators, and clinical specialists and patient experts. But now the document is for public consultation and it would very much help to hear from patients and their families with myelofibrosis with their views. It will really help to get the decision revised...

You can be part of this consultation by completing the short Appraisal Consultation Document on the NICE website -

Please note the closing date is VERY soon.

3 Replies

Cheers Maz. The site info sure makes interesting reading. I sent a letter to a Dr Clayton of Derbyshire to thank him for his submission coz as a GP with Myelofibrosis he really is in a position to give a robust account of how the drug has helped him get his personal and professional life back on track. .


Maz, do you know what the situation is in Scotland regarding this?


I live in Scotland and am trying to access ruxolitinib for post ET MF. It appears that the Scottish Medicines Consortium (the Scottish Equivalent of NICE) has not considered the drug yet and it is not even on their website as a drug to be considered in the immediate future. The Scottish Government have put aside a £21million fund for orphan drugs for rare diseases but on reading the small print it is only for drugs that have been declined by the SCM. That potentially means months before we have access to it. There have been trials for Ruxolitinib in Glasgow and Edinburgh and my haematologist is getting in contact with one of the research leaders in Glasgow to see what the position for me is - but I have not had any good news yet! I will update if/when I hear something.

I have said my piece to NICE but we are a very small voice in the dark as potential consumers with regard to this. I do not feel I have enough qualification to answer each question in their Appraisal Consultation Document but I just said my bit "from the heart"!


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