Log in
MPN Voice
3,465 members5,179 posts

Important opportunity to comment on NICE draft decision re Ruxolitinib for MF patients

THE DEADLINE IS TODAY - TUESDAY 10 NOVEMBER - for commenting on this decision by NICE, please can you all do your utmost to comment and urge NICE to approve Ruxolitinib for patients with MF. Thank you.

Dear all

NICE (The National Institute for Health and Care Excellence) is in the process of deciding whether to approve ruxolitinib for patients with MF (myelofibrosis). A draft of the decision is available via nice.org.uk/guidance/indeve...

A team has been involved from MPN Voice and our concerns are that at present it seems ruxolitinib may only be approved for high risk patients. We have concerns about this since the approved indication is for spleen reduction and symptoms regardless of disease stage. The clinical trials have shown that patients with intermediate risk disease benefit in these aspects but also importantly gain years of life with this drug.

This decision is based upon cost not upon effectiveness of ruxolitinib which NICE agrees with.

This is your opportunity to comment on this via this link:


You will need to register for a NICE account in order to contribute your comments - this is an easy process, when you have clicked on the above link you just need to click on the Sign In button and follow the instructions to create your account or sign in if you already have an account.

Clinicians and nurses will do the same but the voice of patients and their families is a very important one.

Please do so by 10th November.

Thank you.

MPN Voice

6 Replies

I have been one of the two expert patients at both of the NICE appraisals of rux. At the first it was apparent from the first words spoken it would be rejected. Also they would not let Prof Claire Harrison attend, the UK's expert on this drug.

This time the atmosphere was welcoming but it is clear the cost is the main stumbling block.

If you have or know anyone with MF please write to NICE immediately as Mon is the cut off date.

Rux improves length of life and correlates both with the greater daily dose and the greater total life time dose so only allowing it to be given to symptomatic patients classified as high risk ie the most severe who have the smallest life expectancy is perverse.

These are people who have the lowest platelet counts and white cell counts and thus can only tolerate the smallest doses for the shortest times.

It is clear rux is being regulated on total cost.

It is also likely that Novartis will offer NICE a reduced cost for greater access/useage/sales. NICE has no precedent of accepting this ever but for an orphan drug every letter counts.

I have been on rux for 3 years and can truly say it had has a miraculous effect on my life . I could hardly get through the day but this summer rode the Big Zipper, climbed the Via Ferrata Extreme at Honister and cycled from Bristol to reading in 4 days.

I met Jedi Reject as a result of going to NICE for the first time.It didn't work for him so he has taken the gamble of BMT which is a massive risk. I hope he is doing well. There are around 400 people taking rux in the UK right now. It doesn't work for everyone but tell me something that does.

Please write to NICE why they should approve this drug if you have or know anyone with this disease. If not for yourself then for anyone who will get MF in the future.


Thanks for posting this most important item Maz, , and I echo the comments of my friend Skodaguy about the general effectiveness of Rux across the piece. I have scanned the latest NICE submissions and reading between the lines even quickly you can see where the thrust is, being as both Maz and Skodaguy say for high risk MF which in itself limits cost. Whether Novartis will offer a deal based on such reduced patient numbers I don't know.

There is a very strong argument for the drug to be extended to at least intermediate MF to maximise it's effectiveness over time and give those individuals a fighting chance and save them or at least delay them having to go down the BMT route which is indeed a high risk strategy. MF is a difficult nut to crack.

Also we have to remember that the future of any Cancer Drug Fund is in jeopardy in these uncertain times. Each box of 56 15mg tablets that I was on at 2 per day cost over £3000. Which put each tablet at around £58. However there are some cancer drugs / treatments that are much more expensive. And what price extending life?. In the US where the drug was developed the insurance companies pick up the bill, I am convinced this keeps the price artificially higher than it need be but if the company significantly lowers the price for the UK market then it will be in trouble. But it is the best drug currently available for MF and maybe PV.

Ruxolitinib worked for me in terms of general wellbeing and alleviated some of my other symptoms. It was only my spleen size that didn't reduce, but nor did it increase as far as I'm aware. I don't know if there is any evidence as yet that longer term use can reverse MF marrow fibrosis. But as I said before it should be made available to all MF patients with the classic symptoms. And as we know PV/ET can transform to MF.

I would urge any of you guys out there to have a look today, register and give it your support. . Cheers Chris


I forgot to add to that War And Peace submission that NICE see BMT as a viable treatment choice perhaps over drugs for 'healthy' intermediate MF without acknowledging the very high risk and the significant overall cost of the initial transplant and the follow up which I have read is in the region of 150 - 250k.

Let's face it in years to come without a cure I can see all terminally ill, those with incurable life limiting conditions, severely disabled etc, being isolated and living like Lepers. I'd best shut up or the powers that be will be wanting me to stand in the next election. . .


I am not living in the UK (but from Scotland) and live in Australia. My comments probably won't be taken into account, but it's important to say that I've been on Ruxolitinib now for 2.5 years. Not able to have a transplant as I was too ill, Ruxolitinib was provided on compassionate access. I was considered High Risk at the time. This drug saved my life, no question. And my quality of life, although compromised, is better than it was. I was facing the end of the road, so there was nothing to lose. The Australian equivalent of Nice (PBAC) have rejected Ruxolitinib for the last three years, but earlier this year they changed their mind, based on a price adjustment from Novartis (too expensive). Early August, Novartis said they were looking forward to the imminent announcement from our health minister that it would be listed on our PBS schedule (Medicare). Still waiting, but I thinkit's been approved for intermediate and high risk myelofibrosis patients. It would be interesting to know if Nice have seriously looked at the Australian situation with regard to Ruxolitinib. Here's hoping it goes through for you all. Jeanette


Yes I'm taking ruxolitinib ,I did have PV .but after a bone marrow exam last August I have now progressed to Mpn ..Jak poss ..been on the Majic trial for 2 years in August last ,,but not any more .i now get ruxolitinib on the NHS ,,as I'm developing on now ..

If nice don't finance this drug for us ,,is this good bye twinkly. I wonder ?? Oooooow

That's not good news is it ?? Twinkly. Xx


Hi Twinkly, i to get Rux on the NHS via the cancer drugs fund and i understand that regardless of future decisions around availabilty on the NHS as a prescription Or removal of either the cancer drugs fund OR removal of Rux from the list of drugs available on it, that if you are already on it then you will be continued to be provided it for however it proves to be effective.



You may also like...