Training Wheels

When the news about the IOM contract first broke on August 27th, I was at my training for FDA Patient Representatives. Here is my much delayed write-up of that training.

Every year, the FDA Patient Representative Program holds an in-person orientation for the new representatives. With the help of my husband, who drove the car and then pushed my wheelchair around the hotel for two days, I was able to attend this year. Patient representatives are called upon to serve on FDA advisory committees that are considering drug and device applications. I expected a series of dry presentations, but the meeting was much more than that.

“Survivors, fighters and advocates”

photoMy fellow representatives are, in a word, extraordinary. There were many cancer survivors, some of them having beat the disease three or four times. There were patients with multiple sclerosis, Crohn’s disease, Parkinson’s, ALS, alopecia, pulmonary fibrosis, and cardiac disease. There were two parents who have young children with short bowel syndrome and liver damage. I met an extraordinary woman with thalassemia, a rare genetic blood disorder affecting only 1,000 in the United States but many thousands more in other countries. Another woman has mitchondrial myopathy and told me that many patients with that disorder are diagnosed with CFS somewhere along the way.

It was weird to have my disease on my name tag, but I quickly began to see it as a badge of honor. One of the gentlemen with ALS commented that this was “a room full of survivors, fighters and advocates.” Every patient in that room had suffered, and many had looked death in the face. But every single one of us emerged with determination to do something positive. Every patient was there to help their communities. Almost all of us had founded or served on the boards of organizations dedicated to finding answers for our diseases. Many of the patients able to work have made advocacy their careers.

There were several presentations by veteran patient representatives, but the most inspiring was by Matthew Sharp. Matt shared his perspective on HIV/AIDS activism, particularly the evolution of the relationship between activists and FDA. The patient representative program exists because of HIV/AIDS activists, and we have a lot to learn from how they have participated in the drug development process as well.

My one regret about the meeting is that I was not more prepared to introduce myself and ME/CFS. I was one of the first patients to speak during introductions, and I wasn’t sure how much time I could take. I realize now that I assumed most people in the room would know that ME/CFS is a serious disease, but that turned out not to be the case. I had more chances to address it during the meeting and with individual attendees, but I should have had my “this is ME/CFS” elevator speech ready to go, and I missed that opportunity.

The Boring Stuff

There were some technical and mechanical presentations, as I expected. The director of the Office of Health and Constituent Affairs introduced us to the program and to FDA. Over the course of two days, we heard about the drug and device development life cycles, the history of FDA, Medwatch, the orphan drug program and minority health issues. There were key presentations on the extensive conflict of interest clearance process for serving on an advisory committee and how to review the hundreds of pages of information to prepare for a committee meeting.

An key goal of the training was to help us understand the importance of the perspectives we bring to advisory committees and how to contribute to those discussions. Patient representatives are not expected to understand the scientific or statistical minutia of drug applications. Instead, we can bring the instincts, questions, and perspectives of patients and families. Our views are needed on the committees, and we should not be shy about sharing those views. Several representatives talked about the value of asking questions that the scientists wouldn’t think to ask. The bottom line was that we should not feel intimidated by all the scientists and data. FDA wants us there because of our unique perspectives. It was very clear from the FDA employees attending the meeting that they recognize and advocate for the value of patient perspectives at multiple stages in decision making.

What Next?

While some patient reps may be called to serve on advisory committees in the near future, I will probably be waiting a long time. As far as I know, there are no treatments for ME/CFS moving quickly through the pipeline and approaching the final phase of approval. At this time, there is no formal way for me to impact ME/CFS drug development within FDA. I think that the trend is moving towards more participation. For example, the Patient Focused Drug Development meetings are an attempt to capture patient perspectives on specific diseases (ME/CFS included). Within FDA, the need for clearing conflicts of interest are a barrier to swift engagement of patient representatives at meetings with drug developers. But the FDA staff members told us that they are actively pursuing ways to incorporate patient perspectives earlier in the development process.

I was not the only person feeling frustrated about that. Many of the representatives were critical of FDA on this point, and repeatedly asked FDA to be creative in harnessing the expertise of patients. Two of the representatives who attended the meeting have recently created a Facebook group so that we can all stay in touch. The representatives at the meeting had such intelligence and determination that I think we can find ways to harness our collective contributions to achieve practical change. I am certainly committed to doing so.


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18 Responses to Training Wheels

  1. The FDA has no plans of helping CFSers. –> Never have. Never will.

    Just Google “Who owns the FDA?” and you will understand why this insanity has become our reality.

    If you need proof, just read Neenyah Ostrom’s book “50 Things (and 50 More Things) about CFS.”

    When will CFSers’ ever learn that fighting oppression does not happen in a meeting? It does not happen on a webinar. It does not happen sitting at home surfing the web.

    CFS — a politically-defined SYNDROME — is not science. CFS is not even a disease. Syndromes do not even belong in the International Classification of DISEASES (ICD). CFS is 100% political paradigm. And fighting political oppression happens in the streets.

    Solution: Implode the ICD-codes for CFS and AIDS. Without access to ridiculous $ham $yndrome codes disguised as diseases, doctors would be forced to properly diagnose their patients with a legitimate DISEASE. CFS & AIDS would cease to exist. Problem solved.

    • Jennie Spotila says:

      If I didn’t believe that FDA could or would help us, I would not waste my limited energy serving in this capacity.

  2. Ecoclimber says:

    Hi Jennie
    You took apart the IOM SOW to inform ME/CFS patients
    of your concerns

    Take a look at this an tell me if it is impossible based on VA GULF WAR SOW to clearly demonstrate a casual connection

    • sufficient evidence of a causal relationship between an exposure and a health outcome,

    This statement of work has been approved by The National Academies governing body and has been included in the contract between the IOM and the study sponsor. However, in general these statements of work do not detail the specific approach to be used to complete the work, allowing the committee to use its expertise to identify the best approach.

    and compare it to the ME/CFS IOM SOW

    The committee is never going to find a casual relationship between an exposure and a health outcome. We don’t know, let alone a commitee would be able to determine exact causual infectious agent. Thereby the committee will be required to lump everything as an associations of chronic multi-symptoms. Correct?

    The main question I am asking is whether the process they used for transfroming the GWS into CMI will be the same process they will use given our similiar symptoms none of which demonstrates a direct causation to our illness? What do you feel will be the percentages of the IOM redefining ME/CFS into CMI ?


    From reading the testimony the SOW is just a guideline that can be deviated from. That to prove that ME/CFS is not CMI according to the IOM Direct Causation

    • Jennie Spotila says:

      Eco, I’m going to answer your comment over on the Statement of Work post because is it more relevant there.

  3. Jennie Spotila :If I didn’t believe that FDA could or would help us, I would not waste my limited energy serving in this capacity.

    Therein lies the vast difference between someone who understands law (i.e., you) and someone who understands Wall Street (i.e., me).

    The question remains: “Who owns the FDA?”

    Be (as) well (as possible).

  4. Robert Morley says:

    Quoting hyperbolic books and blogs that are generally regarded as conspiracy theories probably isn’t going to gain you a lot of sympathy. How about instead of pushing your own agenda, you stick to provable facts and try to stay mostly on topic. If you can’t do that, I’m sure I won’t be the only person to say that I’d appreciate it if you didn’t post at all.

  5. JohnnyD says:

    Jennie Spotila :
    As far as I know, there are no treatments for ME/CFS moving quickly through the pipeline and approaching the final phase of approval. At this time, there is no formal way for me to impact ME/CFS drug development within FDA..

    Jennie, your statement is disappointing. I think you need to advocate from the position that pandoraorg. net expressed in this PR. ME/CFS experts are advocating from this position, most of them not publicly, and patients need to join in. Ampligen has been in trials for ME/CFS since the late 80’s – only those who have the resources and the strength to move can access. At the FDA meeting in April, Ampligen responders asked that it be moved from rheumatoid arthritis to the anti-viral division. Adcom vote 8 to 5 for safey and 8 to 5 against commercializing. Even if ampligen only helps a small percentage that is relief to hundreds of thousands. The FDA _does_ have the power to move this ‘stuck” drug forward.

    Rituxmab is in trials in Norway. Why don’t we have a trial in the US?

    I could go on and on, but I’ve made my point. We need a consistent and constant message for the need for treatments.

  6. Jennie Spotila says:

    Ok, here I will remind everyone to keep disagreements civil and non-personal. For the most part, posters have followed my comment policies, despite how high emotions have been running in the last month or so. Let’s keep it up. I generally take a hands off approach and let comments through. I would like to continue to do so.

  7. Jennie Spotila says:


    Thanks for the post, JohnnyD. I should clarify.

    I agree that we have to demand more from FDA. Holding that one meeting was not enough. FDA was scheduled to hold a conference call with advocates on October 16th, but that will be postponed if the government doesn’t get back to work soon. There should be a report of the second meeting day from April coming too, I think. What I would like to see is guidance from FDA to developers on endpoints and outcome measures. I think that more could be done on qualifying biomarkers and patient reported outcomes with FDA, too. Patients can have a role in all of that.

    FDA cannot do anything more on Ampligen at this time. They declined to approve it. The ball is now in Hemispherix’s court. They can conduct more studies, reevaluate their data, etc. Procedurally, they have to take some action in order to bring it back to FDA again. That’s not a value judgment on my part. Those are the regulations. The question actually came up at the training I attended, and Dr. Sandra Kweder gave this same answer.

    Regarding Rituximab, there have been attempts to raise funds for a trial here in the United States. I know of several investigators who were interested in conducting a trial but abandoned the idea for a variety of reasons.

    Regarding the pipeline, there are two new treatments starting to move through the pipeline. I can’t comment on either one, since what I know came unofficially. But there is movement. Also, I cannot advocate on behalf of particular products due to the conflict of interest and ethics rules. As a potential member of future advisory committees, I have to be careful about advocating for particular developers or products or I could be “conflicted out.” I am permitted to advocate on behalf of patients as a group, however.

  8. Jennie Spotila says:

    I have revised my comment guidelines because of the exchanges between @lemonfoundation and @Robert Morley.

    @lemonfoundation, I will not allow bickering, nor will I allow unnecessary repetition of the same point or argument. Therefore, I have not approved your most recent comment. Let’s move on.

  9. [Edited to delete a personal attack. My blog, my rules. I have a clear comment policy. Comments that abide by that policy are allowed through moderation, even if I don’t agree with them.]

  10. lemonfoundation :
    [Edited to delete a personal attack. My blog, my rules. I have a clear comment policy. Comments that abide by that policy are allowed through moderation, even if I don’t agree with them.]

    Your relationship with CAA is well known and documented.

    How is that fact a “personal attack”? (so you allow this personal attack about me based on your beliefs without allowing others to judge for themselves?)

    If you review the facts, you’ll see that you moderated a post, and then changed “your rules” after the FACT.

    When will anyone in this community ever take any responsibility for their actions?

    Censor. Censor. Censor. It’s the new american, and it profoundly disturbs me to no end.

    I won’t be posting here any longer. I’m actually defend you in forums in the past, but you lost my respect.

    Be (as) well (as possible).

    • Jennie Spotila says:

      I let this comment through in the interests of transparency. As I said earlier, I had hoped that my warning to you and Robert would be enough. I changed my rules because of the exchange between you, so that it would be clearer that repetition was a cause for moderation in some cases. When you objected, I edited your comment because of my longstanding policy against personal attacks. I prefer to moderate comments rather than ban individuals completely. You are welcome back any time.

  11. Ess says:

    Everyone is trying so hard–and we are all sooooo ill . . .

  12. Robert Morley says:

    @Jennie Spotila
    My apologies if my post was harsher than it should have been. I will be the first to admit that I have little patience for anything around the topic of ME/CFS that isn’t based on hard facts.

  13. Gabby Klein says:

    Thank you Jennie for being our representative there and for reporting back to us.

Comments are closed.