A few weeks ago I did a post on using a Citizens Jury, as a potential way to deal with the emotive issue of a lack of access to HSCT as a potential 1st-line treatment for active MS in the UK.
A Citizens Jury can help sort out the disconnect between the needs or implied needs, of the MS community, and the position of the NHS and/or the HCPs in relation to HSCT. A Citizens’ jury is when a group of people from the general public (not people with MS or vested interests) decide policy on behalf of pwMS and HCPs; in other words, citizens decide if the NHS and/or HCPs are right to withhold an effective treatment from pwMS based on its costs and/or safety profile. At the moment most HCPs feel HSCT is too risky to be a mainstream treatment for MS, whereas a large and increasing number of pwMS don’t.
The following is the results of our survey. I hope the MS Society, NHS England, ABN (Association of British Neurologists) and other stakeholders reflect on these results and consider putting together a Citizens Jury to deal with this thorny issue.
As a pwMS I agree in principle. A positive outcome would be as positive for me as others. However…
Here comes the But, this is a great sounding idea in principle. It is very populist. I mean, who could conceive of a not-guilty verdict?
This falls under the same heading as calling for a referendum on important topics that affect every single inhabitant of the British Isles, and then some. We forget how relatively insignificant we in the MS community are to the greater public.
And yes, before anyone wonders, I voted for remain. As stupid and oversimplified as the question was, one of the many problems. It was a Citizen’s Jury in all but name and as we saw, it served to give a platform for ill-informed and undereducated individuals, and a chance to push an agenda that was best left in history. Any reasonable and rational Brexit arguments – and there are a few that can be made, though personally, I remain unconvinced – were lost in the noise of the loonies.
A citizen’s Jury would say that we no longer trust in experts who ought to be leading the intellectual charge. (I know that some of you are, though many are sitting back – ABN, that’s many of your members). This is not the same as determining someone’s guilt in a binary way. The issue is far more complex, nuanced and beyond the grasp of most of us.
If there were to be a so-called ‘democratic’ process like this (populist and undermines democracy really) then it ought to be on a topic far less specific and far more accessible to people so it is worthwhile participating in the process instead of leaving the supposedly independent Jury to decide based on evidence that is challenged and argued over. If it is decent research, provable, replicable, methodologically sound and so on then it really ought not to be left to laymen to decide over. After all, gravity is just a theory, no? Should there be a Citizen’s Jury over that>? No. Silly idea. However, we have recently seen the results – globally – of what happens when issues are made into populist causes.
We need to have the courage in our system, and really trust the experts. Making policy by a People’s Vote, Twitter Polls, Citizen’s Jury, Social Media Echo Chambers, whatever name one chooses is proving to have very poor outcomes. If you have enough money you can and will side-step all of this. For the vast majority of us, we’d like a decent reliable system that produced robust, evidence-based guidance and didn’t move at such a glacial pace. That is what annoys most people is the incredibly slow pace of progress. If cancer drugs can be fast-tracked and there are ways to have other drugs accelerated through the approvals process then perhaps we need to examine the system that proceeds so incredibly slowly. NICE may indeed have helped budgets and evidence-based Rx but it has grown to such a general morass in some areas that decisions cannot keep pace with progress.
Uh oh! You said the ‘B’ word! 😱
Take cover everyone! Grab tin hats, to the bunkers!
If you read it properly you’ll see that at no point say a B word. I was extremely careful not to!
What you overlay onto it says as much about you 🙂
Any reasonable and rational Brexit arguments
That’s a whopping great B word!
Oh yeah. Dammit. I failed as it was my intention to write without saying the pervasive bloody word.
I want an editor again. I think I’ll fall back on blaming my MS. It usually buys me a little sympathy.
AUTOLOGOUS HEMATOPOIETIC CELL TRANSPLANTATION FOR TREATMENT-REFRACTORY RELAPSING MULTIPLE SCLEROSIS: POSITION STATEMENT FROM THE AMERICAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION.
(…)Review of recent literature identified eight retrospective studies, eight clinical trials, and three meta-analyses/systematic reviews. In aggregate, these studies indicate that AHCT is an efficacious and safe treatment for active relapsing forms of MS to prevent clinical relapses, MRI lesion activity, and disability worsening, and to reverse disability, without unexpected adverse events. Based on the available evidence, the ASBMT recommends that treatment-refractory relapsing MS with high risk of future disability be considered a “standard of care, clinical evidence available” indication for AHCT.(…)
https://www.ncbi.nlm.nih.gov/pubmed/30794930
But is this a cop-out? It says if you don’t respond to anything we are willing to give it a go. They should have asked should we bother with the current DMT and give everyone HSCT?
I suspect with this you would have had alot of fence-sitters, as opposed to endorsing something that seems obvious to endorse, as we can all ready the recent evidence. Why schlep everyone to an international jolly to discuss something that the majority surely agree on? The panel was made up of people who in principal believe in the approach, but would they dare say that we should use thie approach instead of a pharma treatment?
Yes they recomemend it as a “standard of care” as an treatment option but do not provide recommendations on preferred methods for mobilization and stem cell collection, manipulation of the graft, cell dose, conditioning regimen, or post-transplant supportive care. Surely all important if you want people to adopt best current practise. It says I think what you do is reasonable…Maybe this means they can come back for another meeting abit like the “Same time next year”
Long term sequels from HSCT.
My neurologist says stem cell transplantation is justified only when the disease would otherwise kill the patient – cancer I guess. She refers to studies that clearly show that the chemotherapy employed, even in a non-ablative procedure, would significantly speed up the aging of tissues and cause infertility, vascular hardening, osteoporosis and weakens lung function. So her opinion is that HSCT immediately would induce infirmity related to old age even in a middle aged person. Is this correct? Should people whom are newly diagnosed with a mild lesion load submit themselves to HSCT?
Your neurologist is incorrect. Haematologists are the experts in this field, not neurologists. There is more residual damage from Lemtrada than there is from HSCT (Dr Burt is actually on record referring to Lemtrada as ‘liquid HIV’). On the subject of infertility, this is what Dr Majid Kazmi has to say about it:
“For females that don’t regain hormone function post-HSCT, this is treatable with daily medication. The risk of experiencing infertility in non-myeloablative HSCT is age dependent with females below age of 30 unlikely to become permanently infertile but this risk increases with age over 30.
Anyone wishing to preserve their fertility should plan ahead in case a later IVF procedure is required. HSCT will not prevent a woman from having a healthy pregnancy or from bringing a baby to a healthy full term delivery.”
Do we take the word of the UK’s leading practitioner of HSCT for MS or the misinformation of a neuro who probably hadn’t even heard of HSCT 5 years ago (I say that with confidence given the fact that our neuro asked us to spell it…)
SINDRE – This is the same argument deployed by the people who choose not to treat MS with drugs, choose not to vaccinate their children etc. It seems that there is always the one-off doctor who is rolled out to agree with the anti-evidence brigade. This sole source, by virtue of having a medical degree, is then used to prop up the idea that so-called ‘mainstream science’ is somehow being masterminded by a small cabal of evil money men who are locked on an island fortress, rubbing their hands at the thought of all the filthy lucre and manipulating everything to suit their evil ends.
To digress slightly:
The valiant fight for alternative medicine is being led by a small band of tumeric swigging Internet Warriors, unencumbered the boring necessities of trivia like actual evidence. They are driven by gut feeling, hearsay and a sense of righteousness from resisting what in the 1960s was called ‘the man’.
Simply put; alternative medicine that actually works is called medicine. The idea that most senior management in drug companies could organise their efforts to actually manage to collude in any meaningful way is risible. 1/2 of them are great and 1/2 can’t find their own backside with a torch and a map.
If the evidence is overwhelmingly in favour of HSCT (versus the risks of leaving MS untreated or partially treated until the disability worsens) then why shouldn’t people be offered it sooner as a viable option?
To me, that comes across as a fairly typical scaremongering response from a HCP who hasn’t dealt with enough post-HSCT patients, or read as many trial reports as would be necessary to make an all-encompassing statement like that.
Bias is a very difficult thing to manage, and we will all hold biases depending on our circumstances. Before I’d heard of HSCT, my partner had been rapidly declining in health, and climbing in EDSS. When she received HSCT in London almost a year ago, her EDSS would’ve been 6-6.5. Her mobility had been vastly impeded, her speech and vision likewise negatively impacted, along with a myriad of other symptomatic issues.
11 months on, and whilst the road to recovery has hardly been smooth, my partner is back to work, able to do Body Pump classes (which even as a fairly regular gym goer and obstacle race participant, I struggle with) and is almost unrecognisable from the person she was becoming due to the speed at which her MS was progressing. Both my partner and I are now involved in a direct sense with a HSCT supporting charity (AIMS), and we have encountered so many people with similar success stories. In fact, the stats from the clinical trials bear out at around 80-85% success for HSCT, in that no further progression is seen in MRI scans and disability. Many people do, as the author points do, benefit from an improvement in symptoms. The goal is to simply stop the progression, but with many people actually recovering ability, surely that is a win on a much bigger scale. The currently available DMTs only hope to slow progression at best, not stop it cold.
The unfortunate truth is that HSCT is so hard to come by, and the fight to get close to it so draining, that many people don’t have the strength to fight against the HCP in their way whilst also fighting the MS itself.
Something needs to be done to give more people the option and opportunity to access the treatment itself. People can then make informed decisions. The parallel made by someone else to Brexit is actually damningly accurate, with even the MS Society going onto live tv a few short months ago (BBC Breakfast, in November ‘18 I believe) to quote HSCT as an “experimental treatment” that should only be considered if a patient has only months to live. HSCT is basically a bone marrow transplant, so has actually been around for decades. It has also been used to try to try MS for almost 20 years, so it is certainly not experimental, new, or any else of the sort.
Good question. However no point me answering this as I do not have the capacity to offer HSCT but the issue of fertility is a real.
Long term sequels from HSCT.
My neurologist says stem cell transplantation is justified only when the disease would otherwise kill the patient – cancer I guess. She refers to studies that clearly show that the chemotherapy employed, even in a non-ablative procedure, would significantly speed up the aging of tissues and cause infertility, vascular hardening, osteoporosis and weakens lung function. So her opinion is that HSCT immediately would induce infirmity related to old age even in a middle aged person. Is this correct? Should people whom are newly diagnosed with a mild lesion load submit themselves to HSCT?
AIMS thinks that this would be a very valuable group. Our trustees will discuss and respond shortly; as the world’s first HSCT charity it would make sense for AIMS to be at the forefront of the setting up of such a group.
Think Dave Bexfield has some really good sense to say about HSCT:
https://shift.ms/msreporters-expert/dave-bexfield
We need a global petition to make all therapies first line (of course including HSCT). A patient has to have the fundamental right to have the control over his/her life that radically changes with each of the treamtments available. From the choice to have a once and for all treatment to having a treatment with the least side effects, this should be a patient’s and not a Health Administration or doctors choice.
Not having had another chronic condition – one is enough, thank-you – I don’t know whether this MS-centric world view is so common to other chronic conditions. There is a narrative that we are all s0 hard done by and that one ought to battle, fight, resist the condition and append the #MSWarrior tag to everything. I get the feeling that the virtue signalling is high amongst some.
I struggle with the idea that we are somehow especially significant over, say, Lupus sufferers. Imagining for a moment that MS started to be treated differently in the eyes of many then firstly we’d be shot down, secondly we’d set the ’cause’ back and thirdly, we could get a bad reputation.
From a government payer perspective, we are part of a larger overall cost. That is all. We are not individuals to a beancounter, we are a cost. It doesn’t make the individual unsympathetic, but it does mean that they can’t treat everyone as a special case.
This is different when the press starts to roll with a story. Cancer – esp breast cancer or anything paediatric – certainly attracts what appears to be a disproportionately lenient treatment when it comes down to QUALY’s. If I had cancer or whatever the ice-bucket thing was about (ALS?) I’d be thrilled as an individual. More of the cake for me.
As a community, we either need to make a conscious decision to manipulate the media to get a relatively greater share of the pie thus inevitably disadvantaging someone else somewhere (I’d say that most choices come down to this when infinitely reduced) or there is a focus on efforts to improve the speed at which the wheels in the adminisphere that is NICE turn to give to the deserving based on clinical need.
I daresay that the primary reason HSCT is not a first line choice is it as much cost-based as a justified clinical fear of rubbing people out.
FWIW…
@Dominic Cost is definitely not an issue here. Lemtrada and HSCT are much cheaper than conditioning DMTs, HSCT is even cheaper than Lemtrada. There are several studies about this economical aspect. So if anything, cost is an extra benefit (try to be more concise 😉 )
Concise:
A v v bad idea to make complex desicions via populist methods. There is ample evidence of this producing unintended negative consequences.
There are vocal elements of the MS community that seem to thrive on and become defined by a strange blend of a victim and warrior narrative. I think it is counterproductive.
The more cost is politicised the more it becomes a short term thing. Whole life cost of HSCT versus upfront administration and immediate follow-up is different. Short term I imagine it seems pricier, especially when NICE starts modelling it (beyong my knowledge area – cost per QUALY stuff)
Drugs only seem to be treated differently – inequitably – when the PR around them looks as if it stands to harm politicians. Call me a cynic.
Well… much as I hate to say this on a blog run by Neurologists, there is a far simpler solution.
MS is NOT a neurological disorder. It is a ‘disease’ of the immune system that has CNS damage as one of it’s main symptoms. It is a blood disorder and should be treated in exactly the same way as it’s recalcitrant cousin; aka. leukaemia/lymphoma
Transfer all of us to the care of a Haematologist specialising in bone marrow transplants.( Sadly the Neurology auto-immune budget needs to transfer with us).
Haematologists are used to treating patients that may die during their procedures. Neurologists are used to being unable to help their patients – think Alzheimer’s, Parkinson’s, epilepsy, strokes – if these patients die the Neurologist is not too blame. Consequently they don’t have the tools in their tool box to deal with HSCT for MS and we as the patients are short-changed by their fear.
When the Panorama programme on HSCT for MS aired in January 2016 the ABN very quickly put out a statement. Initial it was accessible to anyone and as I read it, I became more and more angry. It was a semi-hysterical knee jerk reaction to something they neither understood, nor were willing to support. Sadly that position statement was quickly moved behind a firewall and I will forever be annoyed that I didn’t screenshot it!
We don’t need a citizen’s jury. We just need our care transferred to the Haematology Department.
PS. And who cares what the MS Society thinks about the patients taking control? They are already coming to the new cutting-edge AIMS charity for information!
MS is NOT a neurological disorder. It is a ‘disease’ of the immune system that has CNS damage as one of it’s main symptoms.
Is that actually known? Relapses are not the disease anyway…
Well, my brain would disagree and my neck. I have aggressive MS but not progressive. I had enough trouble with Tecfidera, which killed my immune system. How dare Dr Burt call lemtrada liquid HIV? He’s an LDN doc.
He’s not a neuro or a haemotologist.
Im so so sick of moronic ‘specialists ‘ over the years that think they have the answer. In fact , I’m absolutely incensed with rage. First we had diet is the answer, as promoted by MSUK. Then we had CCSVI , which doesn’t cure MS. Now we have to discuss a treatment that is for advanced cancer. it’s available to entitled Americans . You want it? Well, it’ll cost you. Don’t look for support from MSUK. They don’t support drug therapy. They should not be allowed at the table.
Dr Richard Burt is a haematologist and immunologist – he knows what he’s talking about when it comes to HSCT and Lemtrada. He’s not an ‘LDN Doctor’ at all. Furthermore, HSCT has been used to treat MS for over 20 years now. In the UK it’s available on the NHS (for free) if you meet the criteria. In America, insurance has covered the procedure for those on the MIST trials or being treated compassionately by Burt. There is a growing HSCT community – over 3,000 people with MS and Autoimmune disease have now been treated. I can tell you with absolute certainty that the vast majority of those people are not ‘entitled Americans’. Yes, it should be made more accessible, but most of those people have had HSCT because they fought tooth and nail and / or fundraised. You’ve said yourself that Tecfidera decimated your immune system, yet you find it difficult to believe that Lemtrada could do that – even when that’s come from a leading immunologist / haematologist. Finally, MS-UK have actually been very interested in what’s happening with HSCT, I’d say far more so than the MS Society – they did a 4 page spread on the treatment in the January edition of New Pathways magazine. Don’t write off HSCT until you know all the facts.