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FDA Workshop on Drug Development for CFS and ME

Discussion in 'General ME/CFS News' started by Ember, Feb 21, 2013.

  1. Ember

    Ember Senior Member

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    2/20/2013 Update
    Enid, taniaaust1, SOC and 2 others like this.
  2. Ember

    Ember Senior Member

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    The FDA “Alternative Approval Pathway...Intended to Address Unmet Medical Need” proposes that a drug's “safety and effectiveness would be studied in a smaller subpopulation of patients with more serious manifestations of a condition. Such a pathway could involve smaller and more rapid clinical trials than would occur if the drug were studied in a broader group of patients with a wide range of clinical manifestations.”

    How will the FDA distinguish in this workshop between CFS and the smaller subpopulation of ME patients with more serious manifestations of the condition?
    taniaaust1 likes this.
  3. Tristen

    Tristen Senior Member

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    Good news...appears they really did listen to all those emails recently.
  4. lnester7

    lnester7 Seven

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    Please make sure to include OI drugs in the discussion! it has taken me out of bed.
    taniaaust1 and Valentijn like this.
  5. Ember

    Ember Senior Member

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    I saw How to Survive a Plague this week. As David France says in this interview, “It was a primer. It really establishes a paradigm for activism.” Their successful FDA encounter is documented below (from about 06:30 to 13:00):



    http://surviveaplague.com/news
    meandthecat and SpecialK82 like this.
  6. SOC

    SOC Moderator and Senior Member

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    Unfortunately, there is unlikely to be any distinction because ME is not a recognized illness at all in the US medical system. It simply doesn't exist. In the US, for anything official, you have CFS. Period. Annoying as hell, but that's where it is right now.
    Jarod, Nielk and Kati like this.
  7. Ember

    Ember Senior Member

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    Clearly the FDA is aware of the lumping or splitting issue, though, as is shown by the use of both CFS and ME in the workshop title and description. During the Teleconference between FDA and Patients/Patient Advocates on September 13, Dr. Sandra Kweder said:
    The FDA knows, of course, that an inclusive frame of reference doesn't work if you're trying to identify a smaller subpopulation of patients with more serious manifestations of a condition.
  8. SOC

    SOC Moderator and Senior Member

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    Well, I certainly won't argue that there's a massive amount of ignorance and lack of basic common sense in the way the medical authorities address ME/CFS in the US. :)
  9. Ember

    Ember Senior Member

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    In How to Survive a Plague, AIDS activist Peter Staley is shown having to take the research initiative:
    When drugs weren't approved by the FDA, public opinion changed:
    The parallels with our situation are striking, and the “inside/outside” strategy makes a lot of sense.
  10. Shell

    Shell Senior Member

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    I still think the biggest barrier to us getting attention is that 'They' insist we aren't dying of this. The wazzak (I refuse to call him a doctor) who dx me went on and on about how no one dies of ME - they die of something else.
    It took my brain a while to unwrap that.
    People don't die of AIDS or Flu either. Nearly every person who died in the 1918 fle pandemic died of pneumonia, and AIDS patients die of cancer or other opportunistic diseases.
    Sadly I think we need to highlight those who have died of opportunistic infection or heart disease caused by ME.

    We somehow need to show that like flu or HIV whatever causes ME is a door opener to all the other crap we are forced to live with or die of.

    The other major disadvantage that we have over those who are hiv+ is that we are ill from the get-go and many of us are very ill indeed. HIV+ people tend to be quite well for the beginning and therefore have the energy for a fight.
  11. Purple

    Purple Bundle of purpliness

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    This point - that we can become severely ill virtually overnight and stay that way for many years or even decades - is in my opinion something to be highlighted in advocacy.
    alex3619, justinreilly, BEG and 4 others like this.
  12. taniaaust1

    taniaaust1 Senior Member

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    I hope the illest of our communities can get representives to go to this meeting in support of them and their needs.

    I too think OI drugs is very important to have something approved for ME/CFS patients. A few of the prescription drugs which really have helped me re the ME are Clonazepam, Temazapam for sleep (not taken at same time as other benzo), Clonidine (can help multiple ME symptoms) and Florinef.
    Ember likes this.
  13. lnester7

    lnester7 Seven

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    Drugs that have helped me the most: Midodrine, florinef, inosine (equivelant to imunovir that is not FDA approved) Sleep Meds: in my case trazadone.

    I have an extremely good testimony how I went from bed ridden to work on 3 days. If somebody is going to the meeting and want to read my statement I am willing to write it. Just PM me.

    (And for those wondering I DO HAVE ME/CFS, been tested by Klimas group, have extremely low NK cell, Low T cells, High B cells and inflammations cytokines. parvo reactivation, casaxie and hh6v). AT is 115bpm.
    taniaaust1, Ember and SOC like this.
  14. Ember

    Ember Senior Member

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    For me, the ME symptom and daily impact that matters most is post-exertional neuroimmune exhaustion (PENE), “a pathological inability to produce sufficient energy on demand with prominent symptoms primarily in the neuroimmune regions.” Pacing helps; GET/CBT doesn't.

    I would welcome any drug that helps to restore my rest/activity balance, such that one hour of rest would reliably support two hours of normal activity. So far, the test-retest protocol provides our best quantitative outcome measure.
    PennyIA and JohnnyD like this.
  15. JohnnyD

    JohnnyD Senior Member

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    Hi Ember, are you talking about Dr. Snell's exercise testing?
  16. Ember

    Ember Senior Member

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    Yes, I am. Staci Stevens presents it here.
  17. Jarod

    Jarod Senior Member

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    Since when did the FDA get interested in developing drugs for CFS? Do they even know what it is? Are they doing the same for autism and other similar diseases I wonder? Do they have an increased budget for such efforts and will there be any project management charts to track results/accountability?

    Do they realize that the best pharmaceutical treatments for CFS have gone up by 500-1000% in some cases? For instance: Stromectol and Invermectin which are anti-parasitic medicines have increased. Invermectin has gone from like $150 for a couple week dose to $1400 suddenly.

    Doxycycline price has shot through the roof and is hard to get. Online price has tripled, but in the US pharmacies has gone up much more and is available in limited quanities.

    Injectable Hydroxyb12 and methyl B12 are extremely hard to get in the US. The price has increased significantly. I couldn't get methyl b12 injectable at all. Hydroxy b12 has gotten expensive and is sold in useless quanities in US pharmacies.

    ------------
    Online pharmacies can be an option for aquiring affordable meds, but the shipping companies seem to be under attack for dealing with online pharmacies. My linked article says for pain killers, but then goes on to say.......
    Not sure if that bodes well for online prescriptions.
    Shell likes this.
  18. Jarod

    Jarod Senior Member

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    Lately the FDA seems to be interested in global regulation activies.

  19. Ember

    Ember Senior Member

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    ME is listed in the ICD-10-CM and it's part of the FDA lexicon. So it must, then, have been deemed to exist. Could it be considered an orphan condition? On the ME and CFS Stakeholder Teleconference in September, Dr. Sandra Kweder told us that the FDA has an “office of orphan products:”
  20. SOC

    SOC Moderator and Senior Member

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    ICD-10-CM is not currently valid and is still subject to change. We're still operating under ICD-9-CM which doesn't have a code for myalgic encephalomyelitis.
    We can hope for future distinctions, but at present they do not exist in the US. At best we get ME/CFS as the broad definition of a single illness, not the distinct ME entity that exists in the WHO classification. Even in the ICD-10-CM ME is coded under Postviral Fatigue Syndrome (not neurological disorders as it is in the WHO-generated ICD documents) and is given as equivalent to CFS.

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