FDA: "The Voice of the Patient” CFS and ME Meeting Report

[Ember]

I just received this email from the FDA announcing the publication of "The Voice of the Patient" CFS and ME Meeting Report:

Dear Stakeholder,

As part of our efforts in Patient-Focused Drug Development, we are pleased to announce the availability of “The Voice of the Patient: Chronic Fatigue Syndrome and Myalgic Encephalomyelitis,” a report that summarizes patient input from the April 25, 2013 FDA Patient-Focused Drug Development meeting and the online docket that closed on August 2, 2013. The report has been posted online and can be found at this website: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm368342.htm.

FDA is grateful to patients, caretakers, and others who so thoughtfully and courageously shared their experiences and perspectives on CFS and ME through the Patient-Focused Drug Development initiative. It is clear that CFS and ME is a debilitating disease that can severely affect a patient’s day-to-day functioning and have a devastating impact on a patient’s life. The variability in the nature of symptoms and in the approaches to treatment is striking. Patients have tried a wide range of drug and non-drug therapies with varying effectiveness, and for some, none are effective.

The April 25, 2013 meeting was the first of the Patient-Focused Drug Development meetings. It allowed FDA to obtain patients’ points of view, in a systematic way, on the severity of CFS and ME, its impact on daily life, and available treatment options. FDA recognizes that patients have a very unique ability to contribute to our understanding of this broader context of the disease, which is important to our role, and that of others, in the drug development process. We share the patient community’s commitment to facilitate the development of safe and effective drug therapies for this disease.

 

[Bob]

Thanks for posting this, Ember.

Has anyone got around to reading this yet? What do you think?
I think it's exceptional. It might be the most comprehensive, detailed and insightful description of my illness that I've ever read. I think it demonstrates a deep insight into the illness, and I was quite moved, emotionally, when reading it.

This is the link for the PDF document:
http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM368806.pdf

 

[Jonathan Edwards]

Thanks for posting this, Ember. I think it would be helpful to have 'FDA' in the thread title, e.g. "FDA: The Voice of the Patient” CFS and ME Meeting Report".

Has anyone got around to reading this yet? What do you think?
I think it's exceptional. It might be the most comprehensive, detailed and insightful description of my illness that I've ever read. I think it demonstrates a deep insight into the illness, and I was quite moved, emotionally, when reading it.

From my different viewpoint, I agree this seems a very positive and helpful document. I just wonder how society has got so screwed up with capitalism that the address is 'Dear Stakeholder'. Is life just being a 'stakeholder'? Is being ill just being a 'stakeholder' (in the illness?)? Maybe the physicians who really do consider themselves stakeholders in illnesses just forgot it's a bit different for patients? But I am being churlish and unfair here. The text transcends the opening and looks to me like a lot of sensible people saying a lot of sensible and useful things. I have only being thinking about ME for 3 months but I can't help thinking this is new at this level - I'd be interested to know what people think.

 

[peggy-sue]

I find this comment a little disturbing:
"It is clear that CFS and ME is a debilitating disease."

Is this just a whole load of bad grammar?
Is it a couple of typos?
Is it another attempt to rename things? (from CFS/ME to CFS + ME)
Does it even distinguish between ME and CFS?

Legal judgements could depend on "something like this" getting through documents.

 

[Bob]

The text transcends the opening and looks to me like a lot of sensible people saying a lot of sensible and useful things. I have only being thinking about ME for 3 months but I can't help thinking this is new at this level - I'd be interested to know what people think.

I don't remember having ever seen anything like this organised by a government agency. I think it's the first time I've ever seen an establishment body (e.g. a government agency or a health authority) actually 'listen' to patients' experiences, in an in-depth consultation, and then describe ME/CFS on the basis of that consultation. (So often, we have to read such nonsense by vested interests who describe an illness that we don't recognise, and the vested interests have a broad influence, and publish a lot of 'scientific' literature.) I'm not sure how this FDA document might be interpreted by someone who hasn't any existing insight into ME/CFS. I've got to read it again with a more critical eye.

 

[Bob]

I find this comment a little disturbing:
"It is clear that CFS and ME is a debilitating disease."

They do explain their terminology:

On April 25, 2013, FDA held a public meeting to hear perspectives from patients with chronic fatigue syndrome (CFS) and myalgic encephalomyelitis (ME) about their disease, its impact on their daily life, and currently available therapies. For this meeting and summary report, the terms CFS, ME, and CFS and ME are used interchangeably in describing the conditions.1
CFS and ME is a serious disease or set of diseases for which there are currently no FDA-approved therapies.
...
1 The terms CFS and ME are intended to be inclusive and make no judgment on the cause of different symptom complexes. At this time, FDA does not endorse any particular disease definition

So they are not making a judgement about whether they have investigated a single disease.

 

[Sasha]

I'd be interested to know what people think.

Part of the process by which this was arrived at seemed pretty extraordinary, to my British eyes. This FDA meeting (which was livestreamed) is the second forum (the other being the twice-yearly livestreamed meetings of the CFS Advisory Committee to the US DHHS) in which I've seen testimony invited from patients, who phone in or give testimony from the floor in five-minute slots and often use it to tell people what it's like to live with the disease. I've seen people on the committees crying during this kind of testimony (though famously in the case of CFSAC, nothing then happens).

I find it hard to imagine either such positive involvement of patients (who are also free to make policy points) or such an open parade of suffering at a UK committee (I'm not sure our culture is geared up to hear it). It amazes me that the latter should be necessary in this day and age - almost as though we have to prove that we are the 'deserving poor'. I always watch it with a great deal of ambivalence - admiration for the guts it takes to stand up and put yourself through giving that kind of distressing testimony on the one hand, and a kind of horror that we should have to parade our wounds like this to get heard.

Possibly slightly off the topic of your question but perhaps interesting, nonetheless.

 

[Lynn]

Okay this is obscure. In the movie Miracle on 34th Street, a judge ruled that Kris Kringle was officially Santa Claus because the United States Post Offices delivered a bunch of letters to him in court. One government agency recognizing the authority of another federal agency. Very far fetched but I wonder if there isn't a grain of truth there?

This FDA document is one federal agency recognizing the seriousness of our illness. Perhaps it will help our cause against HHS if we reference the FDA document in our letters to our congressmen regarding the HHS contract to define our illness.

Lynn

 

[Firestormm]

I just wonder how society has got so screwed up with capitalism that the address is 'Dear Stakeholder'. Is life just being a 'stakeholder'? Is being ill just being a 'stakeholder' (in the illness?)? Maybe the physicians who really do consider themselves stakeholders in illnesses just forgot it's a bit different for patients? But I am being churlish and unfair here.

I had this same conversation with my local NHS Board in 2012 I think. They started calling patients 'service users'. Drove me round the bend! Of course the NHS and Govt. do not want us to consider ourselves 'patients' implying some stationary and obligatory position: they would rather we considered ourselves simply as 'passing through'

I think - trying to be fair - that this email was sent to various people who all had a 'stake' in the proceedings and outcomes. But yeah, I think it's silly.

And I need a break....

 

[Firestormm]

Ember thank you so much for posting this. I have downloaded and only thus far skimmed it, but am on a par with Bob at this point. I think it is very good. Now, if only a similar event could be hosted in the UK by our regulatory players...

 

[peggy-sue]

Thanks for this;
"
peggy-sue said: ↑

I find this comment a little disturbing:
"It is clear that CFS and ME is a debilitating disease."​

They do explain their terminology:

On April 25, 2013, FDA held a public meeting to hear perspectives from patients with chronic fatigue syndrome (CFS) and myalgic encephalomyelitis (ME) about their disease, its impact on their daily life, and currently available therapies. For this meeting and summary report, the terms CFS, ME, and CFS and ME are used interchangeably in describing the conditions.1
CFS and ME is a serious disease or set of diseases for which there are currently no FDA-approved therapies.
...
1 The terms CFS and ME are intended to be inclusive and make no judgment on the cause of different symptom complexes. At this time, FDA does not endorse any particular disease definition​

So they are not making a judgement about whether they have investigated a single disease."

from Bob

well, ok, I did have a good go at reading the whole thing but it was an awful lot of words, it was, honest...
I am still left thinking; "Well, they could still have done better with their grammar."
(sorry. not good brain-wise and very, very grumpy.)

 

[Bob]

not good brain-wise and very, very grumpy.

I know the feeling!

 

[rosie26]

That was a long read. I was impressed. It was a real description of the illness. Taken seriously.


"Stakeholder" very odd. I had a quick look at this thread lastnight and "stakeholder" actually put me off reading any further lastnight. !

 

[Ember]

In light of her recent decision, perhaps Dr. Unger should note that “it was their collective experience with acute, debilitating PEM, which participants called a 'crash' or 'collapse,' that received the most attention at the meeting:”

Many participants also seemed to agree that there are two distinct types of crashes: physical and cognitive. When talking about crashes associated with too much mental exertion, participants mentioned nausea, neck pain, headaches and generally being “wiped out”; as one participant said, “my head feels like it's going to explode...and I really have to have total silence, total darkness.” In contrast, participants associated too much physical exertion with “whole body throbbing” that they associated with muscle pain and inflammation; as one participant said, “like if you had run a marathon....I can barely put one foot in front of the other, barely lift my arm, I jerk.”

Why measure physical crashes with cognitive tests?

 

[Firestormm]

Why measure physical crashes with cognitive tests?

Not sure if that's rhetorical but it could be useful to measure against mental pem - to see if there is a difference? Not that Unger is proposing to overstimulate people mentally and observe: but someone else might and I think should. I know we tend to get very invested in the physical but more attention should be paid to mental exertion and exhaustion/pem.

 

[Ember]

Not sure if that's rhetorical....

Yes, the question was rhetorical. Confounding variables, such as are contemplated here, may wash out her results:

Regarding the OSPAN, there are two sources of data: one from the math portion of the task, which is the processing component, and one from the letter span part of the task, which is the storage component. Patients with CFS did not perform significantly worse on either one of the components, but when looking closer at the results, it is remarkable that especially the letter recall scores are worse in the patients with CFS. The reason why there is only a trend and not a significant difference in the letter span scores is the presence of the high variability between the obtained scores, which is in turn likely the result of the presence of one or more confounding variables (e.g., moment of the day the test was taken, levels of education and intelligence, menopausal state, disease duration).

Notice too how single CPET test results are described in this study:

In a previous study of patients with CFS, Van Oosterwijck et al. demonstrated that postexertional malaise, which is a term used to describe symptom exacerbation as a result of excessive exercise, is triggered by submaximal exercise and self-paced, physiologically limited exercise [41]. Consequently, it is possible to explain the reduced exercise capacity in individuals with CFS by an underlying fear of postexertional malaise. In addition, it is evidenced that patients with CFS report higher ratings of perceived exertion during exercise relative to healthy controls [42–45]. Wallman et al. therefore suggest that the reduced exercise capacity seen in some patients with CFS might be explained by an abnormal sense of effort in these individuals and/or a reluctance to push toward full capacity [45]. On the other hand, we registered a mean RERp value of 1.07 in the patients with CFS, which demonstrates that these patients have cycled to the top of their potential. Likewise, it is well documented that patients with CFS exhibit reduced daily PALs [20–21], and (part of) the cause of this deteriorated physical exercise capacity can possibly be found in the entailed downward spiral of physical inactivity and avoidance behavior toward physical activity.

We have good reason "to get very invested in the physical."

 

[Bob]

The UK's biggest patient charity, Action for ME, has commented on the document.

FDA gives voice to people with M.E.

25 September, 2013

The US Food and Drug Administration (FDA) has published a report on the views of people with M.E. as part of its Voice of the Patient initiative.
Patients were asked questions about the daily impact of the illness and their thoughts on the range of treatments and therapies available to them.
The result is a powerful and thoughtful picture of life with M.E.
Action for M.E. Chief Executive Sonya Chowdhury said, “This document resonates with me on many levels. It offers a close up and very personal insight into the often appalling shared experience of people with M.E. I suggest it is essential reading for anyone wanting to learn and understand more about this dreadful illness.”

http://www.actionforme.org.uk/get-informed/news/your-news/fda-gives-voice-to-people-with-me