. . . to use people as human guinea pigs for orphan or other drug trials when there is little or no hope of the new drug being made available on the NHS.
I was mulling over the events leading up to NICE's recent rejection of Ruxolitinib for NHS use in the UK after months of extensive trialling and evidence gathering both here and previously in the US. After reading on our forum the dilemma currently facing some of you regarding trials I was left wondering if in the light of their decision for MF sufferers is there any point in pressing on with similar trials for ET/PV patients. Or are they just raising false hopes for those people it works well for who might have it free for a the duration of the trial and a bit beyond only to be denied it and possibly start to backtrack.
Then again maybe the drug company will simply pull out of similar trials in the UK.
Or would the potential of wider application and increased end user numbers - there are about 600 MF patients in UK and not even half those would need Rux - have a positive bearing and encourage the drug provider to decrease the price. Or would NICE simply see it as a greater cost the NHS.
These are just some of my random thoughts. I think it's a political hot potato and no doubt greater minds than mine will be mulling over the endless possibilties.
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JediReject
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As I understand it, certainly with ruxolitinib, the drug is available for the rest of your life if needed and you have participated in trials. I guess drugs cannot even get to NICE for approval if they haven't undergone trials with real people. We have seen in the past instances where drug trials have gone horribly wrong so entering a trial should not be done without due thought and consideration. Where the drug companies have people like us with rare, life limiting diseases over a barrel is that when there is nothing else out there they are selling the guinea pigs hope - not the drug.
I have wondered the same thing myself about the trials of ruxolitinib in ET/PV. In fact it makes me quite angry that in future ET/PV sufferers could be in the same position - a too expensive drug that could potentially help but for cost.
Thanks Beetle I thought you would be a kindred spirit - i understand what you say about selling hope rather than the drug. If drug companys keep getting knocked back it will either force them to reduce the cost or they may not even bother with the expensive trialling process in the UK unless they are obligated to.
I don't why it isn't sufficient for US trials to be accepted for consideration here instead of putting a whole new group of folk through it. At least that way hopes and expectations here won't be raised unnecessarily when we have such a rigid cost driven system.
A friend of mine has been on Rux trial and it works well for him and has 'given him his life back', he has 7 months supply left but is'nt sure if he will get a continuance free of charge after that. Surely it's time for the Govt to step in and get all key players together and seek to change the way the whole thing is sorted out for the benefit of all and ultimately save or extend human lives which seems to me to have been 'lost' in our economic climate.
I have been offered this trial (have ET) and couldn't agree more with you, I actually put this question to my hem (he is one of the Doc's recommended for MPNs) why do this when it isn't even available to MF patients, of course many answers to that question... but Novartis will fund the drug for 5 years max, after the trial, then back to best available therapy, it comes back to rare diseases again, funding etc, I feel very lucky however to have had the care I have had on the NHS, in my local hospital, and grateful to be offered a choice, but this drug needs to be available to MF patients 9in my opinion) like you say only a small number would need it, Peg INF is another one Take care
Now really don't know what to do if I'm offered the chance of going on the trial in july. Is it better for me personally not to know if it would help these horrible side effects of hydroxy if I wouldn't get it after the trial . Will I go to develop MF and have seen and felt the effects of a drug that could help a bit . I fear MF and for those people who have it already this must be a horrible time . PV is bad enough for me no further please god . But to answer your question JediReject my friend the real honest answer is money rules the world and if you have enoughof it anything is available . For the the likes of us , we can only have faith that somone somewhere will help us . Take care all and stay strong .
Oh dear! The cynic in me rises to the surface again! Marie95 states that Novartis will only fund the drug for five years post trail. Is it a coincidence that the average survival prognosis for MF is five years? Maybe they are admitting that the drug is not life-extending?? Surely not! Maybe they know that other better drugs will be around in five years. I am counting on that! I see that a doctor in Derbyshire is petitioning his MP about ruxolitinib following the latest NICE decision. That thought had crossed my mind too but not sure if they can actually do anything after NICE has said no - and, collectively even, we are still a very small voice.
Thanks for your responses it's a very tricky area and I guess theres a lot more to it than meets the eye and info' we're not privvy to. I am in contact with the GP in Derbyshire who is making a fantastic effort on our behalfs to get someone that matters to sit up and take notice of this crass system which puts profit or expenditure before people.
In his letter he points up it would cost in the region of 6m a yr to treat MF with Rux and the NHS is currently spending 4m on Homeopathic treatments which aren't medically proven - though its only a comparison and a debate in itself. He put in a tremendous heartfelt submission to NICE because he has MF and couldn't make his Practice commitment prior to taking Rux as part of the trial and he maintains it has transformed his life.
I hope common sense prevails because my only option after HU at the moment is a Bone Marrow Transplant which must cost a few quid in itself and I would welcome the chance to try the Rux which could stave off the need for BMT and if it works for me would hopefully shrink my spleen which is 'uncomfortable' to live with. Cheers
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