This topic contains 8 replies, has 5 voices, and was last updated by 
Mothas 11 years, 6 months ago.
Eve asked an important question in Ali's 'Revlimid' thread and I felt it deserved its own thread so as to give it its own space without hi-jacking Ali's question and replies.
This is the question that Eve asked:
[b] 'My Question is, have the MM TRIALS become more important than the individual patient?'
[/b]
Eve will no doubt expand on the question… but I will make a start.
I assume that Eve is asking if the accent and spotlight on the treatment of MM is increasingly given over to the different trials rather than to the individual patient. We are told from the very beginning of our MM journey (and unlike the X Factor, BGT or The Voice – MM is a true journey) that MM is a very individual disease.
My experience, gathered in the waiting rooms of clinics, Day Case Units, hospital wards and most importantly this forum… and the experiences of everyone that I have had reason to discuss, debate and exchange information with, leads me to a whole hearted agreement that Multiple Myeloma, while sharing a lot in common, is, when all the checks and balances are made, a very individual disease, needing very individual approaches to treatment.
If we can accept that statement as a basic truth then we can start looking at how MM is treated as a very individual disease with each individual patient. Bearing Eve's question in mind we can start with the individual treatment regime for each patient.
Part of the make up of each individual Myeloma patient is the medical histories and conditions that they bring with them. Some may have kidney problems of varying degrees… some caused by the MM, some with pre-existing conditions… both will have an effect of the type of frontline treatment they undergo and what processes, procedures and treatments will be made available further down the line. Others may have heart conditions, some may be diabetic, some may have very little bone damage, others may have extensive bone damage etcetera, etcetera. There are plenty more examples but I have listed the ones above as fairly common examples so as to set the scene for MM being accepted as an individual disease with patients needing different treatments at different stages of their disease progression.
These differing starting points for treatment may well be taken into consideration at the start of treatment but are they truly treated as individual cases? How much flexibility is there in the formulation of treatment plans for each patient? What are the criteria for some patients being offered places on trials, where others are not? Conversely, what are the criteria for some patients being offered maintenance medications with certain other treatments or as stand-alone maintenance for patients in remission?
I was not offered a place on a trial at my frontline treatment stage but there might well not have been any active trials at that time. Are there any here that were not offered a place on active trials, knowing they were available? I was never offered maintenance treatment during my SCT remission although I seemed to have a similar case history to others who were offered maintenance therapy. I had extensive bone damage but for the first three years and nine months following diagnosis, I was not offered Zometa therapy? instead I made do with ?Bonefos? (Sodium Clodronate), in tablet form and not nearly as effective as Zometa. :-/
For those who are not aware, our general treatments are funded by a government organization called [b]NICE[/b] – National Institute for Health and Care Excellence ? [b]NICE[/b] ?provides independent, authoritative and evidence-based guidance on the most effective ways to prevent, diagnose and treat disease and ill health, reducing inequalities and variation.?
At the present time the [b]NICE[/b] approved treatments for Multiple Myeloma are as follows:
[b]Frontline Treatment[/b] (CDT ? RCT ? VCT etcetera) + clinical trials when available
[b]Stem Cell Transplant[/b] – including preparation & stem cell harvest
[b]Velcade & Dexamethasone[/b] – I believe Velcade holds its position here with a primary function as a preparation for a 2nd SCT – for those that qualify, i.e. those who produced sufficient stem cells from their harvest. For those that don?t qualify for a 2nd SCT, Velcade is given as a stand-alone treatment? but as far as I can tell it does not offer a very long remission.
[b]Revlimid & Dexamethasone[/b] ? no remission with this treatment? it is given in tablet form and it works until it relapses. It has a median of circa 30 months but that is extending as further evidence is revealed? with 3 years or more becoming quite common.
[b]Kitchen Sync[/b] ? no official treatment plan and the hospital has to apply for funding for any further treatment. Many people reach this stage, at the relapse from Revlimid feeling fairly good but there is no official treatment in the NICE plan at this stage. Both Kyprolis (Carfilzomib) and Pomalyst (Pomalidomide) have been licensed for use in the USofA but for some reason NICE, and whoever else makes these decisions, insist on the medications being trialled again in this country. God knows why! The US pharmaceutical companies hold worldwide trials, including a good proportion of patients from the UK and Europe, before applying for a license and from what I can tell the US GOVt agency, the FDA (Food & Drug Agency) are very stringent in their processing and do not give licenses lightly. In my opinion there is no need for this trial duplication by the UK? how many people will die while the UK processes these licenses (me for one which is one reason why I?m complaining).;-)
BTW? I got 11 months from Revlimid but I was plagued by a horrible stomach bug call C: Difficule for 9 of those months and I was told by a consultant at another hospital that the C: Diff would almost certainly affected the ability of the Revlimid to absorb. I was taken off Revlimid because my light chains were lingering around the 550 mark (Starting Point 125) and my Platelets had dropped from 140 to 90. I have not given up on Revlimid yet and I am willing to fight my consultants on this point. When my Revlimid was stopped my light chains were at circa 550? in the 8 weeks before starting my Bendamustine treatment my light chains jumped to 2,800. A rise of 425 in 11 months of C: Diff affected Revlimid and a rise of 2, 350 in 8 weeks of no treatment. Ho Hum!
Anyway, the above is offered as background information? My take on Eve?s question is that there does seem to be quite a lot of emphasis on these trials, especially the frontline treatment trials which seem to have been going for a long time without any results being offered for general perusal. The individual has to conform to the financial plans of NICE and the restrictions on ?best individual practice? placed on our medics. The disease might be highly individual but the treatment plans on offer are highly collective? almost Borg-like? with very little leeway for the individual. 🙁
What if I decided that I would like to take my chances with CDT ? Revlimid ? SCT ? Velcade ? (SCT 2 if applicable), then the Kitchen Sync? what would have been the chances of my being able to achieve that order of treatments? Yes, that?s what I thought.:-D
The trouble is, newly diagnosed patients will usually take whatever is on offer? it takes quite a while to become au fait with the ins and outs of treatments, procedures and processes? and the world of MM for new patients would be a far more confusing place without this forum (so thank you MUK for giving us the place and space for us to meet, greet and gather).8-) 😀 🙂
Over to you Eve.8-)
Dai.
Hi Dai
Well you new I would not be able to resist your charms lol (no problems Janet)
I will do my best to put it in some form of order!!!
First Ali,s Revidimide ,the doctor answered her question " if it was your mother" I believe that doctor did the best for patient,but you have to ask yourselves how many doctors would be thinking the same.!!!!
The area I am in,fought hard to get trials, you must also remember its a feather in the cap syndrome,it employs people!!!
I am sure Dai your treatments are all correct,but have to say we have been told after Revidimide maintenance is given.
I honestly believe you do not have a choice of treatment,unless you seek it out and as Tom says become pro active.
They are there!!! But how do you go about finding them!!! Does a consultant sit down with you and tell you what the next treatment is or does he give you options!!! I know what Slims did.
Slim has supplied 7 BMB in nearly 2 1/4 years they know exactly what is going on in his Bone Marrow his Myeloma IgG Bence Jones has been well tracked, they do have data,the patient has no results except what they can glean from any paper work they can get hold of, and sometimes you can ask until you are blue in the face,you still do not get it!!!
Look on the American Site,everyone has all results from test.
It takes awhile and a lot of un answered questions, 5 months for me,to ask what is important to them. The phrase do no harm springs to mind,do I think no treatment in 5 months is doing no harm to Slim!!lots and lots of questions,I have asked for PET scan,told they do not use it on Myeloma patients!!! So many contradictions.!!
After that ramble you may be able to understand why I asked the question.
5 more days and I will phone up and find out the result of the last BMB. 13 days before we see consultant.
This is a complex subject. As a researcher I can see it from the other side too (although not a medic).
Trials are designed to look for specific responses to specifically staged versions of myeloma, for the trial method to be rigorous and consistent only patients with certain symptom and treatment profiles will be admitted.
I've been told that there are no trials available to me at present because of my disease profile. I'm at a large research-based hospital so hopefully I will be eligible at some point.
As far as individual treatment is concerned, there are two ways of thinking about this. Individualised treatment pretty much exists already if you have private insurance. You have speedier access to new drugs and are more likely to be put on maintenance treatments. It's a cost issue and also a post-code issue. Myleoma treatment is a post code lottery.
The second point is that individualised treatment is coming in the form of genetically tailored therapies, that look at the specific myleoma clones active in our systems. It'll be the next step forward in treatment. Hopefully we'll all be around to benefit. 🙂
Hi Tom
Trials offer availability to drugs, that are many times not available under the NHS.
I do not know how researchers work, it would be nice to have some explanation on there work but I do not think that is the issue here.
The criteria for a lot of trials is based around new patients, patients who have not had treatment,as time goes on you have patients like Dai who does not stand much chance of getting on a trial (sorry Dai) ,Keith was a good example,they would welcomed any chance of new drugs coming out of America,last chance saloon it may be. Are the drug company,s scared of the failer rate. I do not know?? May be some one has answers, why are new drugs not available,to people in a position like Dai.
As for GTT,the dater has been collected for years with no results given to people who are supplying the samples,this seems one sided to me.
Would like more in put from people who know a lot more about it than me.Eve
As for new drugs, I believe that trial or no trial, myeloma patients should have the option to benefit from new treatments as soon as they are licensed. What have we got to lose after all.
I was put on the PADIMAC treatment which is a trial at UCLH, without officially being put on the PADIMAC trial.
Don't ask me how that works, I have no idea!
It does seem that it really depends on where you end up having treatment.
Hi all I must say that I do agree about the trials being more important than the person. When Michael was on the Bendamustine trial there were regular phone calls asking what if any side effects he was getting and even when he had to stop treatment (this was because he had very low platelets and the trial criteria stated he could not be given any even though the drug was working,) we had to visit the trial team so he could still be monitored for several months after. But we were then shipped back to our local hospital for CDT.
Even in the early days Michael could not get Velcade or Revlimid because of our post code.we ended up going through our MP. But these drugs were on trial just 20 miles down the road.
Ive learnt a lot about MM and treatments over the years and my opinion is you have to fight for everything
Regards
~Suex
One other point about the trials. I was told I wouldn't be eligible whatever the trial. This is because my kidneys are only working at 20% and the drug companies want the best results from the trials so don't use people like me. This limits my options in the future.
All the best
Scott
Eve's question got me thinking and is the reason for me starting this thread. This thread got me thinking deeper. A few days away from it and I have reached my own personal conclusion… that the trials are being treated with far more importance than the individual Myeloma patient, to the short term detriment of all those patients who fall short of the trial criteria, and to the critical detriment of those that cannot even be considered to even look at the criteria.
It is our consultants who put people forward for these trials… some they know will fulfil the criteria, some they hope will fulfil the criteria and then there is the rest that they don't even bother thinking as candidates for the trial. The consultants are not to blame… the pharmaceutical companies set the criteria and they want the very best chance of getting their product to work. To get the product to work they need their trialists' to be as fit as it is possible to be fit under the circumstances in order to present their findings to the assessment of the national/international agencies who will examine the findings through their own experts before considering the product for licensing.
I asked my consultant why we have to go through the trials again in the UK after being granted a licence by the most stringent agency in the world i.e. the American FAD (Food & Drink) agency. She replied that it is not down to the UK but the European Agency who refuse to accept the findings of the USA FAD. An arrogance I find stunning at the very least. Yes, I believe there should be a top body of European medical scientists and experts who should examine the findings to the minutest level… and that we should be bound by their findings… but only then, if they have doubts, should a European trial be ordered… and not as a matter of procedure.
This way they create jobs for the medics, researchers and committee members but delay by a matter of many, many months the chance for patients to receive life extending medicines. In this delay there are many whose Myeloma will have advanced to a level where the medicines might not properly work and many others who will have died. So yes, trials should be thorough and properly followed to get the best possible results… but thereafter they should be expedited as quickly as possible to reach the consultants and patients.
And this is why the American Myeloma communities are so far advanced than we in Europe. There are a few pharmaceutical companies in Europe but they seem to be far behind the Americans in producing products that truly advance the Myeloma life expectancy. Before Kyprolis June 2012 and Pomalyst January 2013 the last licensed product of any significance that we will recognise is Revlimid 2006… so these products don't turn up that often… but those that do are significant in the world of Myeloma and should be put onto the market and into use as soon as humanly possible… and not delayed without proper and reasonable cause.
Dai.
Dai I agree with a lot of what you say but I think it's fair to say that trials are run for the benefit of research and the patient community that will ultimately benefit them. Trials have to be designed in a certain way to produce useable or useful knowledge about the drugs being tested. If you have too many patients with different types or progressions of myeloma, it becomes very difficult to make sense of the data produced.
However I do think that many of the phase 3 trials are probably not necessary and I firmly believe that alongside the trial regimes patients like us (MM) should have a right to unlicensed or new treatments outside of testing regimes. There was a legal case in the US trying to force the FDA to do this but it failed.
Basically the question isn't 'why aren't more patients allowed onto trials', it's 'why aren't seriously ill myeloma patients allowed early access to new treatments'. We need rigorous testing regimes but that doesn't' rule out early access to new treatments alongside, but not in, testing.
As for the FDA vs European drugs agency, it cuts both ways. There has been a lot of criticism of the FDA because it is quite close to drug companies and there have been issues and cases of undue influence by corporate organisations over the licensing programs. Some of the stuff that the FDA has licensed in the past is unsafe. That's why the EDA doesn't automatically wave through their findings. It's frustrating I agree, but understandable.
Trials aren't of course just run by drugs companies but are carried out by medical researchers at university hospitals such as UCLH. Many of the drugs including kyprolis for example also originate in University research but are developed outside of university by drug companies. This is interesting because drug companies benefit from publicly funded research and then screw the public by making the drugs hideously expensive…
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