A thread on what people with ME/CFS need in the way of service

Hutan said:
There is perhaps the argument that case studies on new drugs won't happen in these 'no off-label prescribing clinics', to build up the evidence supporting a trial.
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It's a thought but the reality is that individual case studies do not 'build up the evidence supporting a trial' unless there is some reliable controlling factor supporting the observations. When we started using rituximab we did 'case studies' in a sense, but we made sure that we were gathering data that could be compared to some sort of control information. In some cases that was detailed pharmacodynamic analysis of objective changes. In others it was historical control data that was good enough to provide high probability that changes were meaningful.

For ME/CFS the problem is that we have nothing much to use of this sort. There are any number of anecdotal stories of benefits of midodrine or fludrocortisone or modafinil but strangely no physician is, deep down, sufficiently convinced of 'building up evidence' to want to do a trial.

A trial with a positive result from any drug would transform our understanding of ME/CFS in a way never seen before - way beyond DecodeME. It would be guaranteed a knighthood in the UK.
 
Perhaps it's just a matter of tweaking the wording to clarify what we are asking for, or at least being able to explain the detail when asked?

We want any new off-label prescribing to be part of a focussed effort to gather good-quality, useful data about efficacy. The expectation is not necessarily that a Phase 3 triple blinded trial with placebos springs from the service fully formed. But years of prescribing a drug followed by a token study of subjective reports from the people who are dedicated users is definitely not ok.
 
Hutan said:
Perhaps it's just a matter of tweaking the wording to clarify what we are asking for, or at least being able to explain the detail when asked?
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Maybe it should tweaked but I think we have had this discussion already and my point was that the wording is actually exactly what it should be. It says nothing about blinding or placebos, just carefully designed controls in a trial - and a case study is a trial. If people do not appreciate the significance then they are probably not the people to be experimenting so would do well to stick to licensed drugs!! I may be being a bit unfair but in EndME's terms the message lands where it should do.
 
Yes, it's just that the target audience isn't just good researchers; we don't want to needlessly put anyone off it because they misunderstand something.

People who are sure that a drug they takes helps them could still be able to get access to it under this model. There is flexibility in what responsible evidence gathering looks like.

To be clear, I'm very happy with what we have and with the communications there have been about it. I just want us to make sure that no one gets the wrong idea about what is proposed. And that opponents have no chance to drum up negative feelings about it.
 
Hutan said:
Yes, it's just that the target audience isn't just good researchers; we don't want to needlessly put anyone off it because they misunderstand something.

People who are sure that a drug they takes helps them could still be able to get access to it under this model. There is flexibility in what responsible evidence gathering looks like.

To be clear, I'm very happy with what we have and with the communications there have been about it. I just want us to make sure that no one gets the wrong idea about what is proposed. And that opponents have no chance to drum up negative feelings about it.
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I strongly agree with this. It may be unnecessary to make it clearer, but there is nothing to lose [from so doing] and potentially a lot to gain. On that basis I think we/you should definitely add some extra explanation along the lines Hutan suggests above.
 
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Hutan said:
Yes, it's just that the target audience isn't just good researchers; we don't want to needlessly put anyone off it because they misunderstand something.
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But I think EndME's point is crucial here. All medics are taught in medical school not to prescribeoff-label. Not prescribing off-label is the norm for other diseases. The stipulation should elicit the mental response "sure, of course". An aspiring ME/CFS clinician is only going to be put off if they have some specific motivation to use drugs off-label. That being the case, if the current wording is off putting they are definitely not the sort of physicians ME/CFS needs.

I think the wording could be improved and am happy to make changes but I have sent this out to the advocacy groups and am keen to see what the response is. I am in the middle of various conversations now. I actually think that it is important to flush out objections to this because they can illustrate muddled thinking. If adherence to NICE is used as the central argument for a new service format then you cannot have off-label prescribing, since the status of off-label drugs is exactly the same as that for GET and the Lightning Process - no reliable evidence base and risk of harm.
 
My only misgiving about the no off-label prescribing point is the issue raised by @Nightsong:
Nightsong said:
A lot of medications used for sleep, pain, headache disorders are technically prescribed off-label - even if there is trial evidence for them it may not be the licenced indication.
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Do we have members who could look into which medications in these categories would not be available to pwME if a strict no off-label policy were adhered to?
 
Evergreen said:
My only misgiving about the no off-label prescribing point is the issue raised by @Nightsong:
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Technically yes, but nobody is actually going to object on this basis. People will object because they want access to drugs for which there is no simple logical extrapolation to ME/CFS. If wider leeway is allowed there is also no way to stop mindfulness and breathing exercises and acupuncture and all that stuff, which some physicians major on.

A policy not to use drugs off-label beyond simple logical extrapolations for things like pain is standard for medical care. It should not be an issue for someone seriously wanting to provide a good service. The option of wider off-label usage has been already been raised in contexts where I think members here would not want leeway. It would not have been raised for pain and sleep meds.
 
Jonathan Edwards said:
Technically yes, but nobody is actually going to object on this basis. People will object because they want access to drugs for which there is no simple logical extrapolation to ME/CFS. If wider leeway is allowed there is also no way to stop mindfulness and breathing exercises and acupuncture and all that stuff, which some physicians major on.

A policy not to use drugs off-label beyond simple logical extrapolations for things like pain is standard for medical care. It should not be an issue for someone seriously wanting to provide a good service. The option of wider off-label usage has been already been raised in contexts where I think members here would not want leeway. It would not have been raised for pain and sleep meds.
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Yes, I imagine the objections were objections I would object to.

OK, so those "simple logical extrapolations for things like pain" would still be possible in these clinics, but we wouldn't be pestered to take Abilify or LDN (if trials are negative).

This is the dream.
 
The other issue with excluding off label is that its off label in the UK. Given the lack of research and the entire political landscape in the UK it will be very far behind places that are accepting the reality of the disease like Germany and the USA. As such drugs may very well get approved there many years even decades ahead of the UK and there will be no money for repetition.

People also have different thresholds for what is considered sufficient evidence to try a medication, while the healthcare system as a whole has a high standard and rightly so we are talking about a group of patients who are often willing to risk death for small improvements and willing to try things on less evidence than in other diseases precisely because there are zero approved treatments. There are going to be major issues with approval given the DHSC is once again driving the rehab model of care.
 
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BrightCandle said:
The other issue with excluding off label is that its off label in the UK. Given the lack of research and the entire political landscape in the UK it will be very far behind places that are accepting the reality of the disease like Germany and the USA. As such drugs may very well get approved there many years even decades ahead of the UK and there will be no money for repetition.
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Lack of research in the UK seems a strange way to put things. DecodeME is far and away the most successful piece of research in recent years. Germany seem to have been stuck in poor quality studies and dubious extrapolations to risky treatments. It is good to see funding there now but my impression is that scientifically they are about 5 years behind. German regulatory authorities have always been pretty dubious in terms of what they approve. The blood clot and antibody stuff is exactly the sort of thing the patients need to be protected from until such time as we have meaningful data.

I don't see any acceptance of the disease in the USA other than as a money spinner for some private outfits. The research there has also been pretty low level. Hanson has been trying hard to get useful data but it still seems a bit disjointed.
 
BrightCandle said:
As such drugs may very well get approved there many years even decades ahead of the UK and there will be no money for repetition.
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I think this is excessively pessimistic. If something really works for MECFS it will be very difficult for the rehabbers to argue against prescribing it, and very difficult for the NHS to make the case that they shouldn't provide it if people who can access it privately are improving significantly.
 
V.R.T. said:
I think this is excessively pessimistic. If something really works for MECFS it will be very difficult for the rehabbers to argue against prescribing it, and very difficult for the NHS to make the case that they shouldn't provide it if people who can access it privately are improving significantly.
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They will jump at the opportunity to present themselves as the saviours of pwME/CFS and conveniently forget the previous neglect. As long as there is a way to save face, they will take it.
 
BrightCandle said:
There are going to be major issues with approval given the DHSC is once again driving the rehab model of care.
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I don't think there will be if the evidence is strong. DHSC doesn't direct patient care.

It might take some time to get NICE approval for a treatment, but that's a different process. NICE needs evidence to assess, and it would take a while for that to accrue. But I don't think the absence of a NICE recommendation or guideline necessarily stops the treatment being used in the meantime.
 
Kitty said:
I don't think there will be if the evidence is strong. DHSC doesn't direct patient care.

It might take some time to get NICE approval for a treatment, but that's a different process. NICE needs evidence to assess, and it would take a while for that to accrue. But I don't think the absence of a NICE recommendation or guideline necessarily stops the treatment being used in the meantime.
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Medication approval by the FDA is typically slightly quicker than by the EMA which is slightly quicker than the MHRA. I think that is the only difference. I doubt it will make much of a difference here, especially in a situation where it would be the first approval of a medication and people might anyways start prescribing before then, be it off-label, via compassionate use or whatever you want to call it. I don't think this will be a genuine hurdle and if it will only make a difference of a few weeks.

There is definitely the situation of off-label use in other countries. But that has nothing to do with evidence. The threshold for off-label medications in Germany doesn't exist. It doesn't have anything to do with higher thresholds, they just said "let them eat cake antidepression pills" to the LC patients, there's no evidence involved.
 
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