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CFS and Ampligen: A Creative Solution from the FDA?

Discussion in 'General ME/CFS News' started by JohnnyD, Feb 2, 2013.

  1. JohnnyD

    JohnnyD Senior Member

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    The FDA is going to hold a little webinar on February 4th an 5th. The following was posted to the federal register on 01/15/2013.
    Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need; Public Hearing

    The Food and Drug Administration (FDA) is announcing a public hearing to obtain input on a potential new pathway to expedite the development of drugs, including biological products, for serious or life-threatening conditions that would address an unmet medical need. The 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. The labeling of drugs approved using this pathway would make clear that the drug is narrowly indicated for use in limited, well-defined subpopulations in which the drug’s benefits have been shown to outweigh its risks. The purpose of the public hearing is to obtain information and comments from the public on the need for and feasibility of this pathway and its potential advantages and disadvantages.


    Full posting here:
    https://www.federalregister.gov/art...-drugs-intended-to-address-unmet-medical-need

    This fits CFS and Ampligen to a "T". We know that members of AdCom were calling for the FDA to get "creative". We know that ampligen did not quite fit the FDASIA requirements (they needed a surrogate endpoint). We know that a large trial would be very difficult, given the nature of the disease, and associated problems.

    I think the FDA may get it. This could change everything.
  2. Sasha

    Sasha Fine, thank you

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    Interesting...

    I hope this doesn't mean a delay in conditional approval of Ampligen, though.
    snowathlete and Nico like this.
  3. JohnnyD

    JohnnyD Senior Member

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    CENTER FOR DRUG EVALUATION AND RESEARCH (CDER)
    FOOD AND DRUG ADMINISTRATION DEPARTMENT OF HEALTH
    AND HUMAN SERVICES

    Public Hearing: February 4, 2013

    Creating an Alternative Approval Pathway for Certain Drugs Intended
    to Address Unmet Medical Need; Public Hearing

    Panel Members

    Presiding Officer: Rachel E. Sherman, M.D., M.P.H.
    Associate Director for Medical Policy, Director, Office of Medical Policy
    Center for Drug Evaluation and Research

    Edward Cox, M.D., M.P.H.
    Director, Office of Antimicrobial Products, Office of New Drugs
    Center for Drug Evaluation and Research
    Issam Zineh, Pharm.D., M.P.H., FCCP

    Director, Office of Clinical Pharmacology, Office of Translational Sciences
    Center for Drug Evaluation and Research

    Diane Maloney, J.D.
    Associate Director for Policy
    Center for Biologics Evaluation and Research

    Abigail E. Brandel
    Attorney, Office of Chief Counsel
    Office of the Commissioner

    Richard Klein
    Director, Patient Liaison Programs, Office of Special Health Issues, Office of External
    Affairs
    Office of the Commissioner

    Creating an Alternative Approval Pathway for Certain Drugs Intended to Address
    Unmet Medical Need
    Part 15 Public Hearing

    February 4, 2013
    FDA White Oak Campus
    10903 New Hampshire Ave, Building 31, Room 1503
    Silver Spring, Maryland 20993
    Each speaker will have 10 minutes for their presentation. There will be 5 minutes
    following each presentation to offer an opportunity for the panel to ask clarifying
    questions.
    February 4, 2013 Presentations
    9:00 – 9:10 am Presiding Officer Opening Remarks
    Rachel Sherman, MD, MPH
    Associate Director for Medical Policy,
    Center for Drug Evaluation and Research
    9:11 – 9:21 am (9:21– 9:26 am)
    Jeffrey Spaeder, Quintiles
    9:27 – 9:37am (9:37– 9:42 am)
    Roger Echols, Infectious Disease Drug Development Consulting, LLC
    9:43 – 9:53 am (9:53– 9:58 am)
    Rebecca O’Connor, Parkinson’s Action Network
    9:59 – 10:09 am (10:09– 10:14 am)
    Jeffrey Stein, Antibiotics Working Group (AWG),
    Trius Therapeutics
    BREAK
    10:14 – 10:30 am
    10:31 – 10:41 am (10:41– 10:46 am)
    Robert Guidos, Infectious Diseases Society of America (IDSA)
    10:47 – 10:57 am (10:57– 11:02 am)
    Paul Huckle, GlaxoSmithKline (GSK)
    11:03 – 11:13 am (11:13– 11:18 am)
    Jennifer A. Jackson, Cubist Pharmaceuticals, Inc.
    11:19 – 11:29 am (11:29– 11:34 am)

    John F. Crowley, Amicus Therapeutics, Inc.
    John R. Kirk, Amicus Therapeutics, Inc.
    Jayne C. Gershkowitz, Amicus Therapeutics, Inc.
    11:35 – 11:45 am (11:45– 11:50 am)
    John H. Powers, George Washington University School of Medicine,
    University of Maryland School of Medicine
    11:51 – 12:01 pm (12:01– 12:06 pm)
    Nicole Mahoney, The Pew Charitable Trusts
    LUNCH Ala Carte items will be available for purchase on site
    12:07 am – 1:07 pm
    1:08 – 1:18 pm (1:18– 1:23 pm)
    David Ross, George Washington University School of Medicine
    1:24 – 1:34 pm (1:34– 1:39 pm)
    Andrew J. Emmett, Biotechnology Industry Organization (BIO)
    1:40 – 1:50 pm (1:50– 1:55 pm)
    Jonathan Sackner-Bernstein, ExVivos, LLC
    1:56 – 2:06 pm (2:06– 2:11 pm)
    Alan Solinger, Pharmaceutical Product Development, LLC (PPD)
    Association of Clinical Research Organizations (ACRO)
    2:12 – 2:22 pm (2:22– 2:27 pm)
    Roslyn Mannon, American Society of Transplantation (AST),
    University of Alabama, Birmingham
    2:28 – 2:38 pm (2:38– 2:43 pm)
    James I. Healy, Soffinova Ventures,
    National Venture Capital Association (NVCA)
    BREAK
    2:44 – 2:59 pm
    3:00 – 3:10 pm (3:10– 3:15 pm)
    Anthony Castaldo, Hereditary Angioedema Association
    3:16 – 3:26 pm (3:26– 3:31 pm)
    Jennifer Yttri, National Research Center for Women & Families
    3:32 – 3:42 pm (3:42– 3:47 pm)

    Brian Rosen, Leukemia & Lymphoma Society
    Mark Velleca, Leukemia & Lymphoma Society
    3:48 – 3:58 pm (3:58– 4:03 pm)
    Sally Okun, PatientsLikeMe
    David Clifford, PatientsLikeMe
    4:04 – 4:14 pm (4:14– 4:19 pm)
    Diane Helentjaris, The American Medical Women’s Association (AMWA)
    4:20 – 4:30 pm (4:30– 4:35 pm)
    Randy Wheelock, Consumer
    Open Public Comments
    4:36 – 5:05 pm
    Closing remarks/Adjournment
    5:05 – 5:15 pm
  4. Research 1st

    Research 1st

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    Thank you for posting this news update JohnnyD. It seemed unlikely the future of Ampligen and CFS would be decided on a Saturday. I am pleased to hear there will be a public hearing, good news I hope for at least a compromise if the drug is not to be approved?
    taniaaust1 likes this.
  5. jimells

    jimells Senior Member

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    This seems like more distraction and delay to me. How many years will they take to 'approve' this new 'pathway'?

    "But you don't understand... we have to follow the Rule of Law..."

    ...except when it's inconvenient, like when it's time to start another war, or kidnap and torture "terrorists", or lock someone up in a military brig without charges or trial, or murder citizens in foreign countries by remote control...
    peggy-sue and ggingues like this.
  6. Lynne B

    Lynne B Senior Member

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    Hi, Johnny D, This sounds like a good thing to do in general. The trouble is, I don't see anyone on the panel to speak for our medical condition. Also, I think the fact that several of us, such as Bob Miller, have already established how much Ampligen has done for them, so I don't know why that would have to be established all over again before approval was given for them to use it.

    regards, Lynne B
    taniaaust1 likes this.
  7. JohnnyD

    JohnnyD Senior Member

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    We'll have to ait and see what Hemispherx says next week. But it appears that ampligen will not get approval this time - it does not fit well with the current approval pathways. The new pathway they are proposing in this seminar, it seems like a very good fit.

    While know CFS rep is on the panel - I'm sure this was set up months ago and the timing would not be good if ampligen is rejected - the proposed new pathway is still a fit for CFS.

    Lynne B, unfortunately the FDA needs controlled trials to pass a drug. The new pathway proposes, smaller, quicker trials.

    They are taling public comments until February 26th. If ampligen gets rejected, which I suspect, we need to convert The Petition drive into a Public Commentary drive to submit as many public comments to the panel as possible.
  8. taniaaust1

    taniaaust1 Senior Member

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    This implies a lot.. it implies they wont currently be able to pass Ampligen as it doesnt mwet the current rules.. so may mean that Ampligen is about to get a big NO .. unless they put the Ampligen approval on hold till they bring this new thing in. As a ME/CFS patient I'd rather see the whole Ampligen decision thing get put on hold till their current rules are sorted out, rather then just a no.

    This new drug ruling will be a very good thing and I think WILL happen. Why I think it will certainly happen is cause it will have all those drug companies behind it.. eager to get their drugs out faster. Money makes this world go round one could say..profit, profit, profit.

    I think possibly it needs to be pushed that this all needs changing fast and without lengthly meeting delays and I think it appears that is exactly what they want to do as that date... is very very soon. Much sooner then I would of thought if they decided to think about changing things... that date (Feb 4th) could of been in 4-6 mths time..but its almost right away..how often are gov meetings arranged that fast? (i bet not often). So I think they are trying to rush to change things so it appears for once the American gov is trying to do something good for us for a change. (I cant believe Im saying this.. I think this is the first time ever that Ive beleived they are trying to do something for us).

    The one big concern I do have thou is why isnt there a ME spokesperson or org who deals with the "severe" patient group..what this new change will be about.. why isnt one going to be speaking at that meeting esp when this new ruling if brought in will help ME/CFS patients greatly? Why are certain patient groups represented and not us??????????????? eg Hereditary Angioedema Association, Parkinson’s Action Network, American Society of Transplantation, Infectious Diseases Society of America, Leukemia & Lymphoma Society (they actually have 2 of their spokespersons talking, so a double time slot)

    Are the gov still trying to "play it safe" and dont want our illness to be seen as a serious one.. not serious enough to have representation for our severe patient group at this very important meeting????. I think some new advocacy efforts are needed right now.. to email those responsible for this meeting and ask WHY arent the severe ME/CFS patient group being ignored at this meeting with no representation for us there. I propose that if they dont give ME/CFS a serious representation for the severe patient group of ME that ME/CFS patients should use that meeting as a PROTEST opportunity to protest how this illness is being IGNORED, with the very sick patients going invisible.

    Please those in America.. please protest outside this meeting if you can if we dont get some decent spokesperson there on severe ME.. (you dont need to be protesting with others to do a protest... whether its just one protestor or many.. we need to protest at that meeting if our illness is ignored). Dont allow this ignoral of ME/CFS patients go on..BE SEEN. (Not long ago I had a laying down protest .. I actually feel asleep during it at a medical conference which was ignoring ME/CFS).

    I hope our well known ME/CFS advocates hear about my idea for a protest there in regards to once again this illness being ignored and will call up a protest there.
  9. Shell

    Shell Senior Member

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    I'm afraid I too think this looks like stalling. When you consider how many vaccines and other drugs get approval without this much faffing about and with far less understanding of how they work.

    Let's take Clozaril for example. It was approved back in my day (1980s) even though it was well known to have truly horrible side effects. It was such a toxic drug that patients on it had to have 3 monthly then 6 mthly bloods taken. We would get a report back from the lab marked as "green" meaning carry on, "amber" meaning things looked bad and "red" meaning patient had to be taken off the drug.
    Lots of patients with intractable forms of pychosis, particularly scizophrenia were put on this drug and it helped a lot of them (though not all by any means)

    And yet for Ampligen the goal posts don't so much move as race around dodging the ball.
    Is this political by any chance? Is this a fear that by approving it the FDA are admitting the CDC were pillocks of the first order and this isn't all in our silly female heads?
    jimells and taniaaust1 like this.
  10. LaurelW

    LaurelW Senior Member

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    Anybody know if this meeting will be webcast and if we can watch it? I didn't see anything about that on the link.
  11. Ember

    Ember Senior Member

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    Who would represent "the severe patient group of ME?" With reference to CFS, how would "a smaller subpopulation of patients with more serious manifestations of [the] condition" be defined?
  12. Waverunner

    Waverunner Senior Member

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    I remember, that NICE approved a Multiple Sclerosis drug although it didn't pass their evidence based requirements. The reason they approved it was, that until then there was no treatment for MS and they wanted to give MS patients at least some form of hope. Maybe this applies here too.
    taniaaust1 likes this.
  13. taniaaust1

    taniaaust1 Senior Member

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    i didnt word that well did I. Ive not a clue but as they are refering to severe "subgroups" of illnesses and approval for just subgroups of illnessses.. how to define those things?? ..which groups would qualify under a policy change and which ones wouldnt. ... that would be up to them.

    Those who arent very sick should they be approved to trial dangerous or life threatening drugs?? The severity of each individuals illness needs to be well weighed up with the risk of the drug. Should someone who has ME/CFS but can still work full time.. be given an experimental drug which kills off one type of cells or carries a very high risk?? At this point of research..where there isnt a lot of current research.. should dangerous drug recommendations be only made to homebound or bedbound patients or to all?. Wouldnt it be irresponsible thou if dangerous drug recommendations without a lot of research behind them were being recommended to those not so sick? (not housebound or bedbound).

    Why should the completely bedridden ME/CFS ones be denied drugs on the grounds it is too much risk (as what is happening now).. as they may be too much risk for one not as disabled by the illness if one weighs up risks verses possible benefits. Currently severe patients get left to like rot.. and cant get the more risky treatments they may need. eg I cant get the saline IVs I need, I'd like an indwelling catheder put in if saliva IVs helped where are this risk wouldnt be worth the risk for many others. ..most ME/CFS info doesnt even mention daily saline IVs. Why? cause the risks" for most" outweigh the benefits. Does that mean that it shouldnt be approved for those who need them? The severe group of ME/CFS which needs more risky treatments currently misses out. There is currently an imbalance going on.

    I'd actually thou also like to see medication approval rather based on what kind of symptoms people get then severity (as I actually think ME is happening in all kinds of severity with some more minor cases) eg those showing more viral symptoms eg fevers, sore throats, swollen glands, coughing etc should have a chance to trial antivirals etc. But anyway.. things need to start somewhere with some meds of different types starting to be approved for our illness. Let us tral things till science sorts itself out. (actually with patients trialing things and the severe groups trialing more high risk drugs.. it may actually help science faster realise what is helping patients and what isnt

    As far as who could represent the sicker ME/CFS group... as I dont live in America I cant say as I dont know the ME/CFS groups there well enough but I do know there is some orgs there who tend to "play down" the serious of our disease and hence wouldnt be very good supporters of the completely bedridden patient group. Here in Australia we have the Alison Hunter Memorial Foundation who represents the very sick group and helps highlight just how severe some ME/CFS patients can be and who has had a big impact on things here over the years... they help educate doctors, have at times run and funded ME/CFS medical conferences etc etc.
  14. taniaaust1

    taniaaust1 Senior Member

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    I think it is certainly politics that one of our representives arent talking at that policy change meeting. They probably dont want to highlight the ME/CFS situation in any way at all.. its an burial.
    Shell likes this.
  15. taniaaust1

    taniaaust1 Senior Member

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    Maybe things could lead then to a group legal case .. group discrimination if they knock Ampligen back . Could enough evidence be put together to show that gov decisions are being made biased towards ME/CFS?
  16. Ember

    Ember Senior Member

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    According to the FDA proposal, “the drug's safety and effectiveness would be studied in a smaller subpopulation of patients with more serious manifestations of a condition.” So the FDA is already looking for serious (not just severe) manifestations of the condition.

    In her FDA presentation at the October CFSAC meeting, Dr. Sandra Kweder said that “a research definition is needed so that we can begin to study things.... The only way you learn is by starting someplace. Clinical therapeutics are not going to be developed until this is done:”
    The ICC already identifies a smaller subpopulation of patients with "more serious manifestations of the condition."
    taniaaust1 likes this.
  17. Ember

    Ember Senior Member

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    Webcast Update:
    Either electronic or written comments will be accepted after the hearing until March 1, 2013
    (https://www.federalregister.gov/art...-drugs-intended-to-address-unmet-medical-need):
    I can't find the docket number for identifying comments.

    Transcripts of the public hearing will be available for review on the Internet at http://www.regulations.gov approximately 30 days after the public hearing.
  18. JohnnyD

    JohnnyD Senior Member

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    Speculation on my part. Regarding identifying sub-groups and those severely afflicted. I think this is what the CDC has been doing with their multi-site clinical assessment. They started this assessment in July, posted to webiste in November, results are due this month or next I believe. From CDC website.

    "Ultimately, this study aims to improve how we measure illness domains of CFS. This may allow patients to be sub-grouped to improve therapy and allow the underlying biology to be discovered."

    This new pathway hearing has obviously been worked on for months, but only posted the the public register on Jan 15. I see lots of positives. Regarding having a seat at the table for this public hearing. I cannot think of another disease, besides CFS, that meets the criteria. The problem with current FDA pathways with such a large population as CFS is the larger phase III trials required. A thousand patient trial of CFS patients would be extremely difficult... for any drug maker large or small. So from the hearings description, IMO, CFS IS the table. The FDA has a stakeholder scheduled for Spring. If they reject ampligen this time around (likely), This gives them a way forward and they might actually get someone to show up at the stakeholders meeting. I mean besides protestors. They need to attract drug makers and they need a way to get ampligen approved with out some long drawn out large phase III trial. It means patients have to wait yet again, but this is a way forward.

    taniaaust1 and Sasha like this.
  19. Sasha

    Sasha Fine, thank you

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    Very interesting!
  20. JohnnyD

    JohnnyD Senior Member

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    The word is that HEB received a 70 page CRL from the FDA late Friday afternoon. HEB is preparing their response/PR which will probably be released this afternoon.
    taniaaust1 likes this.

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