I am very sorry to have to share the news that NICE, the National Institute for Health and Clinical Excellence, has rejected approval of tocilizumab for GCA. Their decision is open to consultation, and we would like as many GCA patients as possible to comment on their website about this decision.
Tocilizumab (TCZ), or Actemra, which has been approved for GCA in the US and Canada, is a relatively new 'biologic' drug, which directly targets IL-6, one of the key 'interleukins' that is a major player in the development of giant cells and the inflammation of the artery linings. The submission for approval was based on the international GiACTA study, which combined weekly infusions of TCZ with a tapering steroid programme. TCZ has been demonstrated to aid tapering in rheumatoid arthritis and reduce the overall 'burden' of cumulative steroid doses. It demonstrated a similar effect for GCA and appeared to cut down rates of relapse considerably.
Here at PMRGCAuk we are disappointed of course, not least because this is the first significant step forward in treating GCA for over half a century! It simply isn't acceptable that patients with GCA should have to languish on yoyoing doses of steroids for years and years, while people with other diseases have a range of alternative drugs to choose from.
NICE have done a (very complicated) calculation and come to the conclusion that TCZ is too expensive, because it won't deliver on 'Quality of Life Years'. One of the parameters of this calculation is their assumption that 'most' patients are elderly. In fact, they have extrapolated from the 'mean' age of onset of 72 the assertion that 'most' people with GCA are over 80! Clearly we are going to challenge this, politely of course, but it would be great if as many people as possible could get involved in the consultation.
To do so, you have to visit the NICE website and register. And read the documents. Then fill in the online consultation form.
We only have till 4 January to do this, before the committee meets again to consider the consultation submissions and make their final decision (which btw won't be reviewed for 3 years). Seeing as Christmas and New Year come in the middle we are really pushed for time.
I'll be posting more on this thread but meanwhile please have a look at this link that gives the clearest yet explanation of what TCZ is and what the fuss is all about.
I am very sorry to hear this as my Rheumy has said I would get TCZ if NICE would approve it. I will certainly register and fill in the consultation form. Thank you for the update.
Yet another example of NICE getting it very very wrong. But it is also a perfect example of the bias patients with both PMR and GCA face in getting a diagnosis "Only people over 70 develop these diseases".
No, we aren't all "over 70" - the average age at diagnosis may have been 72 (that was some years ago) but that means some are a lot younger and they aren't identifying younger patients because they believe it can't happen.
I don't actually believe it should be offered for every patient willy nilly but it should be available for selected patients - can I assume that may continue?
Not as it stands at the moment. But if we are successful in the consultation we can hope that it will be approved for 'refractory' cases, i.e. those who have repeated flares/relapses. I don't think there's a snowball's chance in hell that it could be approved for all cases of GCA. But we wouldn't be asking for that anyway.
That's no different to you buying a mobile phone on a contract and a short time into it saying you've changed your mind. You still have to pay out the rest of the contract.
Actually, the £40bn is over several years and wouldn't go far in the NHS: "When the NHS was launched in 1948, it had a budget of £437 million (roughly £15 billion at today's value). For 2015/16, the overall NHS budget was around £116.4 billion."
Yes, I think so. But in RA you have to have worked your way through the entire list of drugs that are available, starting usually with methotrexate and the other DMARDs and have failed them all. The biologics are rarely the place they start - for obvious reasons. And the argument will be that pred works reliably in GCA to avoid loss of vision when started in a timely manner and tcz isn't going to change that.
In a few years there will be other IL-6 inhibitors available, both competitor drugs and biosimilars, and that will bring the price down a LOT which will alter the arithmetic. So it will then probably become available on the NHS. At the moment tcz is the only one and as such can maintain its price.
So you have to remember that even though the drug is approved for GCA in Canada and USA neither country has publicly funded pharmacare so individual patients may be no better off in North America.
This is disheartening news. I wonder if the decision could be challenged on equality grounds as it is a disease that predominantly affects older, females?
It isn't the end of the world nor the end of the story.
In a few years there will be other IL-6 inhibitors available, both competitor drugs and biosimilars, and that will bring the price down a LOT which will alter the arithmetic. So it will then probably become available on the NHS. At the moment tcz is the only one and as such can maintain its price.
This is directed at RA but the IL-6 drugs are basically for RA and it explains the whole thing quite well - because it isn't just the NHS that has the problem, even the USA has limitations to drug prescribing. Table 2 shows the drugs that came online once the patents expired for their reference drugs in 2015/16 and are being used widely.
I filled in the form last night and emphasized quality of life for Senior Citizens who have paid their taxes and contributed towards the prosperity of this Country.
Is it the case if tcz were approved for GCA the cost would be covered through NHS? It may be true that the drug has been approved in Canada and USA for GCA but the cost will be borne by patients, either out of pocket or through their private/workplace plans (which may decide not to pay) as neither country has publicly funded pharmacare.
It is - the whole of NHS healthcare is free at point of receipt and the NHS is entirely funded through public taxation of one sort or another (by that I mean not only the NAtional Insurance contributions). If you choose to use private health care you would use it to pay for the enitre cost of care for that illness - but no private insurance plan that I know of in the UK will cover chronic illness. They will pay cover of new illness until renewal and then decline to renew - meaning the patient then has to join the NHS queue.
This is a shame Kate. I am in the US and on shot #13. I had PMR and followed with GCA. Have been on steroids for almost 6 years. Nevrgot below 12. Developed many illnesses as a result of high doses of steroids. Also many relapses. In the end it is more costly to treat all the illnesses created by the steroids than Actemra. Am now down to 8mg of prednisone. Just as an example of the added expense I am Taking prolia shots, pre diabetic need 2 eye surgeries, torn knee meniscus which requires a new knee because the tears are not getting any blood supply. I’m starting physical therapy. This makes no sense for the UK not pay For the Actemra . In the long run it will cost them much more. My ESR is 4 and my CRP is 0.029. Never had numbers like that on steroids alone. Wish I could help you. Your website and in particular PMRpro have saved my life. Thank you Kate.
Hello Nap1. I am in the US and have discussed Acterma with my Rheum. He has used it for Rheum patients. I know a person who was on it effectively treating their arthritis, but had to come off due to elevated liver function tests. I went to the government clinical trial site. I found that The Mayo Clinic is doing a clinical trial on Baricitinib. And Massachucetts General is doing one on Ustekinumab. That's Stelara, a drug I hear on commercials for psoriasis. clinicaltrials.gov/ct2/resu...
Thanks Marilyn for that lovely message. It would be really great if you could write to us about your experience on Actemra. If you could email me on kate.gilbert@pmrgca.org.uk I'll be happy to discuss with you how we might benefit from your story.
This decision comes as no surprise to me whatsoever. So disappointing for me, particularly as I was diagnosed with PMR and GCA in July 2015 and have really struggled to get below 10mg of Prednisolone. In recent months I was definitely made to feel that I was failing in some way and reluctantly agreed to take on Methotrexate, something that for obvious reasons I absolutely didn't want, but felt I had no choice (I now have MTX by weekly injection and it is so toxic that if you spilt some on the carpet it would burn a whole in it.... goodness only knows what it is doing to the inside of my body) I have asked to be considered for Tocilizumab, but told that I would have to try all other alternatives first, but it now sounds as if this will never happen either. Of course the focus of this NICE paper was based on money, but in view of the fact that GCA is rare the actual costs to the NHS would have been minimal was clearly not a factor in reaching this decision, or was ignored. As you can guess from my rant that I do feel very strongly about NICE's decision, so will register to submit my views. Isn't it interesting that it has taken so long for NICE to reach this decision and any appeals, which have to be submitted in a matter of a few short weeks, will take 3 years before being reviewed again. No surprise there then!! In the meantime I think we should consider all the most appropriate options available to us that will deliver maximum impact to raise awareness to this cause. We shouldn't have to fight for what is right and fair, but we always have to, particularly when you are of a mature age and put to the back of the medical queue!! The "E" for Excellence in NICE isn't deserved in this instance!!!
TCZ was considered last year in a consultation document for Takayashu's and for GCA. It was approved for Takayashu's - because it is so rare and only suffered by under 50s - but not for GCA. It is identical to GCA histologically - and the argument against GCA was there was no evidence supporting its efficacy. That was done just a few months before the GiACTA trial reported and they said they would reconsider if evidence was put forward. This is their reconsideration.
I do know of at least one person who discovered her diagnosis of Takayashu's had been amended on her notes to GCA. She asked why. Takayashu's is only for under 50s - after 50 it is considered to be GCA she was told. Now I'm sure if she could be found a good lawyer could make hay with that little gem!
"The key difference between Takayasu’s arteritis (TA) and giant cell arteritis (GCA) is the age of the patients affected by the disorders. Takayasu’s arteritis affects younger patients, generally less than 40 years of age, while giant cell arteritis affects older patients, generally over 50 years of age."
So if you are still of childbearing age you merit this new treatment. If you are only a grandmother you don't, despite evidence that grandmothers are crucial in helping to raise the young of our species.
I am not sure that is a good comparison of TAK and GCA.
TAK can really affect the aorta and large vessels off it. I have know patients lose kidneys, need aortic aneurysm surgery, develop pulmonary aneurysms etc with TAK. GCA mainly affects the Temporal artery ( but not always which is why patients with GCA should see Rheumatologists to have the extent of the disease mapped ).
TAK responds to Methotrexate, Rituximab, anti TNF's and Mycophenolate as well as prednisolone. It may be a continuum of a similar disease but the overall disease burden can be much greater with TAK.
Ah well, what do I know? Just quoting a page from what looks like a reputable source. At the moment I have no skin in this game. Don't live in UK, don't have GCA.
"TAK can really affect the aorta and large vessels off it."
So can GCA and my quote about them being histologically identical came from a medical paper. You have joined in the misapprehension that "GCA mainly affects the Temporal artery". No it doesn't but I have yet to meet many patients with GCA who have had " the extent of the disease mapped". Very few patients are sent for PET scanning - but even "only PMR" patients have been found to have inflammation of main arteries (the brachial and subclavian arteries are often involved and the research rheumies do do u/s of them).
says "Aortic aneurysm, dissection and large vessel stenoses are well-recognized complications of GCA and these occur in approximately one-third of patients (5). Until recently, few studies have evaluated the presence and distribution of large-vessel involvement in GCA. Use of ultrasonography and positron emission tomography (PET) in newly diagnosed GCA patients reveal a surprising frequency of large-vessel involvement (6, 7). In addition to aortic arch involvement, vasculitis affecting the LE vessels is often seen on imaging studies"
I would think that the main reason patients with TAK demonstrate such broad tissue damage is probably that they develop it younger, ergo have it longer. GCA probably does more often affect cranial arteries, and not just the temporal artery, but it does affect aorta and other thoracic arteries and the guidelines say GCA patients should be monitored for signs of aortic aneurysm. But if it starts in their late 70s they are less likely to have 10-15 years of unmanaged inflammation causing ongoing tissue damage.
The primary dispute has to be though the concept that TAK affects under 40s, GCA affects over 50s, primarily over 70s. A survey of the patients associated with PMRGCAUK might show a lot of people in their 50s before/at diagnosis - but what on earth happens to the poor sods in their 40s? They fall through the cracks rather effectively don't they? There have been people on the forums in their 40s with typical GCA symptoms but ignored even by rheumatologists with comments of "If you were older I'd say it was GCA" or "Caucasians don't develop TAK" (they do).
I don't disagree with anything you say or the articles you have posted but am struggling to understand why you feel that I suffer from a misapprehension that GCA only affects the Temporal artery when I didn't state that.
One of the questions I always ask patients who phone the VUK helpline with GCA is have they at least had the BP checked on both arms and their pulses listened into for bruits.
I am on record on this page as saying that I don't think that a Chest X ray is sufficient to rule out aortic aneurysms, my own ascending thoracic aortic aneurysm which extends into my aortic arch isn't visible on chest X Ray. I hope the soon to be published revised evidence based guidelines for GCA will provide guidance on this issue.
VUK have a fair few members over 50 who have a diagnosis of and are been treated for TAK and we signpost everyone to Prof Justin Mason as he is the UK/ world TAK expert as we know that quality of treatment varies throughout the UK.
I suspect that the case of the 27 year old in Wales who has was given a diagnosis post mortem of GCA ( which is often quoted on here ) actually had TAK. One of my concerns is that TAK isn't ruled out when patients in the 40's and above develop GCA type symptoms.
As regards the damage done to younger patients TAK seems to present in a different way. Patients get an acute illness with ( sometimes ) raised Inflamatory markers, joint pain, weight loss, fever etc. They then come out of the acute phase and symptoms settle, inflammation and damage continues in the badckground and they then re present with ischaemic or other symptoms casused by narrowed arteries, aneurysms etc.
The average time to diagnosis for TAK can take years.
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Nearly forgot the biggest difference between TAK and GCA is that TAK responds very well to MTX etc whereas the prevailing opinion on this site is that GCA doesn't. 😀
He was 37 - and it was the pathologist who said GCA.
I'm sorry - I extended your comment that "GCA mainly affects the temporal artery" a bit far perhaps - but far too many doctors think that too. There is a very good reason why so many TABs are negative.
In reality, relatively few patients with GCA are offered methotrexate because the doctors are of the opinion that pred is their only option and it is unethical to mess about with other medications.
However - in the newest recommendations I believe it is being suggested methotrexate be introduced in the first month of treatment for GCA as a study suggests that then it does improve outcome while later introduction does not.
Where it is our experience that it doesn't often help is in the very much later stages to reduce doses that are actually aready relatively low. Why not isn't obvious.
That's a really good point - something we should mention in the submission about whether they have considered all the evidence - they haven't considered their own evidence imo.
I am starting Actemra next week. I was on prednisone for 2 years 8 months. I then was on methotrexate but it was not working for me. I have GCA (temporal arteritis.) The price is extremely high if you do not have good insurance. Has anyone been on Actemra that can give me any opinion how it works.
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