NICE consultation on new drug for GCA - we only have till 4 January!

[Polywotsit]

Hi! Do you think you might have a spare half hour between now and 4 Jan? We would really value your support. You may have read that NICE, the National Institute for Health and Clinical Excellence has so far refused to approve the use of tocilizumab for GCA. We feel that there are flaws in their arguments, and have sent our comments to them via the consultation website. If you have or have had GCA and have either -

experience of tocilizumab, perhaps through private practice, or -

suffered significantly from steroid side effects or not being able to reduce your dose -

it would be great if you could send your own comments to NICE via the website. We only have till 4 January to do this. You will need to register, and to read the documents, which to be honest are pretty heavy reading. Here's a link to the website: bit.ly/nice_tcz_gca.

And to help you, here's a link to the text of PMRGCAuk's submission: bit.ly/submissionNICE

It would have impact if you could focus your comments around one of the following points -

a) NICE seem to be assuming that 'most people' with GCA are in their 80s

b) they have approved it for Takiyasu's Arteritis, which is very similar to GCA. In doing so they referred as evidence to a trial with GCA patients!

c) the 'burden' of long term steroid doses in terms of side effects and long term complications is life-changing for patients

d) TCZ targets the cause of the symptoms rather than just the symptoms. It is the first specific new treatment for GCA in over 60 years, i.e. in the lifetime of many people who are now suffering from GCA.

Thanks, and have a joyful Christmas and a healthy New Year!

[PMRpro Ambassador]

Kate - do you think this

academic.oup.com/rheumatolo...

has appeared anywhere in the argument? I don't know whether it would be a plus or minus since it would actually increases the potential numbers of patients!

However, at my recent appointment with Christian Dejaco we agreed that I almost certainly do fit into that central area that covers it all! I have LVV that causes PMR and also has caused cranial GCA symptoms before aborting. I have had "PMR" for 14 years now and been on pred for over 8 years. I was 51 when the symptoms first appeared (including the cranial GCA ones of jaw claudication and scalp pain) but I didn't know what I know now. The GP couldn't get PMR right never mind anything any more complex and I finally got a sort-of diagnosis 5 years into the journey.

It is far too late to establish anything for certain for me now - and I suspect there are a lot of people in a similar boat. There are a lot of young people who are ignored or given a whole range of diagnoses which are wrong - which leads to a lot of morbidity and wasted years at the very least, far worse for many.

[Polywotsit PMRGCAuk team member in reply to PMRpro]

Hi again - I have now seen the draft BSR submission and they are taking the spectrum into account by proposing that TCZ is approved 'just' for those with repeated relapses, major side effects from pred, and LVV involvement.

[PMRpro Ambassador in reply to Polywotsit]

Which truthfully is what it SHOULD be. And on that basis they could add in PMR!!!!

[Polywotsit PMRGCAuk team member]

You're right about the potential! However, NICE can only consider approvals for what the pharma company has applied for (i.e confirmed GCA ). But in future........

I've discussed this spectrum concept with the authors and long thought that it accounts for why some like yourself have such a tough time for years and others have a more straightforward story. We've had to wait for the evidence from imaging, haven't we? But it feels like a breakthrough. Maybe if TCZ were approved then people on the middle part of the spectrum could be treated too, eventually.

Merry Christmas! Have a piece of panettone for me!

[PMRpro Ambassador in reply to Polywotsit]

No panettone I'm afraid (the wheat-free ones are pathetic) - but I may have a pizza for xmas dinner Will that do?

What I am so pleased about with this paper is that it is what I was getting at in my question to Baskhar at the Centre for Life "do" when the DVD was launched. I've been saying it for a LONG time.

[jinasc in reply to PMRpro]

I well remember that question on Wed 12th May 2010 and our first actual meeting. We have travelled far in those 8 years since then, and with Kate I go further back.

We will win the LVV, perhaps not in my lifetime.

[Jackoh]

Thank you for this Kate. I have registered with NICE and have trawled through the documents. You have supplied what I was going to get round to suggesting or asking for - which is some more guidelines on what to submit. There was so much to trawl through and the points you have made and the submission made, makes it a lot clearer to know what to submit as an individual. There isn't a lot of time and at a very busy time of year,one might even think that it was planned that way!!! So I would encourage all those who are able to participate to do that. It could make a difference!!! Jackie x

[Polywotsit PMRGCAuk team member in reply to Jackoh]

Thanks Jackie - you're right, it's a mountain to climb. I understand that the key PMR and GCA specialists are getting mobilised to make their own submissions. So it will help so much if we have the patient voice there too.

[Hidden]

I researched this drug, and there is a class suit in the process going on as it has caused several deaths! I am not sure how accurate this info is, but as for me I will not consent to any treatment unless is a 100% clear that is safe.

[PMRpro Ambassador in reply to Hidden]

If you wait for a treatment that is 100% safe you will wait a long long time! No paracetamol, ibuprofen - anything that has a desirable EFFECT will very likely also have a less desirable SIDE EFFECT.

As I understand it, the class action is because it was marketed for RA without any mention of the cardiovascular side effects. Which made people believe it was safer than other biologics used in the same scenario. It isn't any less safe than the others - but the selective presentation was misleading. It isn't because it was believed to have "caused" the deaths.

[Polywotsit PMRGCAuk team member in reply to PMRpro]

Well put!