1. Patients launch a $1.27 million crowdfunding campaign for ME/CFS gut microbiome study.
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No Longer Naive in the Ways of The Beast
After having lived for years with ME/CFS, Jody Smith learned there's more to this beast of an illness than she realized, and that what might help one person may not help others ...
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Jenny Spotila on the upcoming FDA meeting:

Discussion in 'Action Alerts and Advocacy' started by Sasha, Mar 20, 2013.

  1. Sasha

    Sasha Fine, thank you

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    Very interesting post on Jenny's blog, as always and here's some key stuff from it below. Some important links for you to take action haven't copied across - please anyway read the whole thing on her site:

    http://www.occupycfs.com/2013/03/19/showcase/

    The patient session on April 25th will be watched closely by more than just ME/CFS stakeholders. If I were an advocate for ALS or gastroparesis or narcolepsy, I would pay very close attention to this session as the test case for how future PFDD meetings will be conducted.

    The same is true for drug developers and researchers, including those who do not currently work on ME/CFS. This meeting has the potential to be one of the biggest stages our ME/CFS community has ever had.

    [...]


    We have four hours on April 25th to describe how ME/CFS affects us, list the many treatments we’ve tried, and make the case for immediate investment in developing new treatments. We will have the microphone for half a day, and we will be watched by FDA, HHS, drug companies, and advocates for other diseases.

    What we say at the meeting will help us or hurt us. Showing drug developers and FDA that there is a market for new treatments, and telling them which symptoms to target and how to measure improvement, will help us. Complaining about the meeting or the long list of things that FDA and other agencies have done wrong will hurt us.


    This meeting is a tremendous opportunity for us to be heard. Let’s give voice to the symptoms and treatments we deal with. Let’s fill the public docket with comments. Let’s carefully craft our answers to the questions FDA has posed to us. If you can attend the meeting, sign up to be a panelist or commenter. If you can’t make it, sign up for the webcast and submit written comments. I’ve explained how the signup process works and the questions FDA is focused on, and I’ll be doing more posts in the next 5 1/2 weeks. The CFIDS Association is planning some webinars to help you prepare as well.


    This is our moment. FDA wants to hear what we have to say about our disease and the need for treatment. We have to get this right. Let’s use this opportunity to teach people about our disease. Let’s show the world what we need, and that we’re willing to participate constructively in the process to get it. There’s no way to know when (or if) we’ll have this opportunity again. Let’s bring our A game to this showcase event.
  2. snowathlete

    snowathlete

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    Thanks Sasha for posting this. I just registered for the meeting and sent a link to the content team as I'm sure PR will be covering this.
    Sasha likes this.
  3. jspotila

    jspotila Senior Member

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    Thanks for sharing and linking to my blog, Sasha. I will be doing more coverage of FDA-related issues over the next few weeks.
  4. lnester7

    lnester7 Seven

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    Please make emphasis on your lab abnormalities, this is very important.
  5. caledonia

    caledonia

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    Why reinvent the wheel - the CCC or ICC has a list of symptoms, the Karnofsky scale shows how badly it can affect us, PatientsLikeMe has a list of the many things people have tried. Here's a list of tests that will show abnormalities - http://www.name-us.org/MECFSExplainPages/TestAbnormalities.htm#Tests_for_Abnormalities_in_ME/CFS

    The rate of disability is 25%. I'm not sure of the source of this - a study done by Leonard Jason? His studies provide the best statistical info about us.
  6. Sasha

    Sasha Fine, thank you

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    The questions aren't about what symptoms we have or how severe they are but what symptoms are most important to us to get rid of. That kind of survey is increasingly being done in patients with various diseases - it's an important shift to taking into account what's most important to patients.

    In the context of the FDA workshop, drug companies will want to know whether particular symptoms are important enough to us for them to put money and effort into looking at their products for us.
    Snow Leopard and urbantravels like this.
  7. urbantravels

    urbantravels disjecta membra

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    Once again, Sasha makes the point I like to think I would have if my brain were working right! (Lots of fog at the moment...) The point is not reciting a list of our symptoms. The point is that we need to be able to demonstrate, among other things, which symptoms impact us the most and where current treatments are failing us.

    Of course we desperately need treatments that will treat the central aspect of our disease - I'll call it "debilitation" rather than just "fatigue." And there are currently NO treatments that target that core reality of our disease. But we also have many symptoms for which treatments, in theory, are already available - the problem being that so many of these symptomatic treatments fall short for our specific needs.

    I suspect, for instance, that a large percentage of those of us who have pain have also had a lot of difficulty controlling it with meds. Some pain meds don't work, or give us bad reactions, or stop working over time, or doctors don't want to prescribe us certain things, etc. Anecdotally we've all heard plenty on this particular topic, so we can guess there would probably be a high percentage of problems out there. The surveys are a way to attach hard numbers to that "probably."

    Same goes for sleep treatments and the like. There are quite a number of sleep medications on the market, but what percentage of us do you think would report that we have tried numerous things and most (or all) of them don't work?

    Seriously, we need a betting pool. Can't pay off on the bets without good survey results.
    jspotila, ggingues and SOC like this.
  8. Sasha

    Sasha Fine, thank you

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    Actually, that's the major point and I forgot it altogether, urban! I forgot the survey asked me what I'd tried and whether it worked. Nothing works for me for any of my symptoms except a sleep med works, a bit. Drug companies are going to perk up at that. A gap in the market, with millions of customers in that gap.

    Also: the more of us who fill in the questionnaire, the keener a market we look and the more we emphasis again our numbers and how active we're becoming in putting on pressure.
  9. jspotila

    jspotila Senior Member

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    I think it is critically important to tie symptoms and biomarkers and attempted treatments - all three, wrapped up with a bow and tied to function. Symptoms/biomarkers/treatments all impact our daily functioning, and that's one of the things the audience needs to hear. It's not fatigue or pain or PEM by itself that is the real issue - it's the impact of fatigue/pain/PEM/etc on our ability to function. FDA wants to hear about the impact of symptoms and treatments. Biomarkers - such as abnormal lab results - are not meaningful by themselves (in the context of FDA), but only as they relate to functional improvement or lack thereof.

    The ultimate goal in clinical trials is to meeting the endpoints defined at the beginning of the study. If I create a drug that I believe makes PEM less severe, then an important endpoint in the clinical trial of my drug is demonstration that PEM is less severe. And drug makers must define that endpoint and how they will measure it at the outset of the clinical trial. The choices of the endpoints and how to measure success are critical, and we can help shape that at this meeting. (I plan to post more about biomarkers and endpoints some time before the meeting.)
    Snow Leopard and Sasha like this.
  10. lnester7

    lnester7 Seven

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    In my case Midodrine+ florinef took me from bed ridden to working full time in a matter of Days. So I think is doable if you find the underlying reason for you particular case. unfortunately most are not so lucky.
  11. jspotila

    jspotila Senior Member

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