The upcoming FDA Drug Development Workshop for ME/CFS will be a showcase for our disease and our patient community. We must prepare now to bring our A game on April 25th-26th.

FDA first announced this workshop in the summer of 2012. Originally, this meeting was described as a one day meeting and the draft agenda focused primarily on scientific issues including clinical trial design, outcome measures, use of off-label drugs, and risk/benefit analysis. At the same time, FDA began the process of developing Patient Focused Drug Development (PFDD) meetings for a short list of diseases, including ME/CFS. PFDD is mandated by the PDUFA V law. FDA is required to hold 20 disease focused meetings to collect patient input on symptoms and treatments. FDA selected 39 candidate diseases and held a public meeting (and comment docket) to collect public input on the selection of the final 20 diseases. I wrote about that meeting, and four ME/CFS advocates offered comment there.

Our efforts to put ME/CFS on the final list were successful. Early this year, FDA decided to combine the planned ME/CFS meeting with the PFDD process. A half day patient comment session was added to the agenda. We will have four hours on April 25th to teach FDA and drug developers about ME/CFS, our symptoms, and our current treatments.

Dr. Theresa Michele (Medical Officer Team Leader, Center for Drug Evaluation and Research) told me that the decision to make ME/CFS the premier PFDD meeting was very significant. She said it sends an important signal to policy makers and drug companies that FDA believes that drug development for ME/CFS should be a priority. The Pink Sheet Daily (an industry publication) said,

The decision to make CFS/ME the focus of one of the highly coveted “disease of the quarter” meetings under the reauthorized Prescription Drug User Fee Act cements these conditions as a test case for incorporating patient input into the drug development process for conditions with a high unmet need.

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.

Whenever I write testimony or do an interview about ME/CFS, I first ask myself “What is the most important thing my audience needs to know?” As we prepare for the April meeting, we should be asking ourselves similar questions: What do we want FDA to learn about what it’s like to live with ME/CFS? What is the best way to measure whether a treatment is helping us? What do we want drug developers to learn about the symptoms we want treated? What image do we want to show the public? If these diverse audiences learn only one thing about ME/CFS, what should it be?

Some members of the ME/CFS patient community are voicing harsh criticism of FDA, claiming that the “right” experts have not been invited. Based on the names that have leaked, this is not true. There are expert clinicians, researchers, and patients on the speaker list (full disclosure: I’m on the list). We all have preferred experts – maybe our own doctors or researchers we’ve followed for years. But this meeting is not a popularity contest. We should judge this meeting based on the full agenda and speaker list, not whether our personal favorites made the cut. Update March 20, 2013: a draft agenda and speaker list is now available (pdf link).

We need to stop whining and complaining, and start preparing for this meeting. 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.


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7 Responses to Showcase

  1. Pingback: Draft agenda posted for the FDA Meeting April 25th and 26th, 2013 | Speak Up About ME

  2. Joe Landson says:

    For the record, I am not a drug spammer 😉
    I plan to go and testify at some level. My dilemma is this: I usually bring a note of levity into all my public evidence, mostly from fear that being serious will bring out the rage, anger and frustration that I actually feel. Should I continue keeping it funny for this very important meeting, or throw for long yardage, so to speak? Advice welcomed.

    • Jennie Spotila says:

      Joe, if I hadn’t actually shaken your hand I’m not sure I could believe your “I’m not a drug spammer” comment! Actually, can you deliver that line like Nixon’s “I am not a crook?”

      Anyway, I think it’s really important to answer FDA’s questions seriously. Given that this meeting will be watched by so many people not familiar with ME/CFS, and therefore not familiar with the rage and frustration we feel towards the government, levity and sarcasm could be easily misinterpreted. I hope that everyone – patients, FDA, researchers, drug developers, policy makers – can come to the table with an attitude of constructive problem solving. Let’s try to find solutions to the problems that plague clinical trials. Pharma has to see that this area can be lower risk, but if the patient population gives the impression that it is not interested in working constructively, that is one more element of risk they have to deal with. Furthermore, if there is a general impression that ME/CFS is actually an emotional/mental illness and patients inadvertently confirm that stereotype, drug developers have no reason to get involved. It will be CBT, GET and antidepressants for all.

      As difficult and painful as it is to speak honestly about this disease in public, I think we all need to keep it real in our comments. Hope this helps!

  3. Eric says:

    While I think there should be more representatives of different ME/CFS organisations on the list, I fully agree with this

    “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.”

    I think it’s good we don’t accept things we don’t agree with, but it’s also very important we are prepared to work seriously and constructively with the people and organisations that can help us and that we need, such as the FDA, researchers and drug companies.

  4. Steve says:


    It is not fair to say people are “whining and complaining” when the best CFS expert in the world, the one who discovered the Incline Village outbreak, and who uses an array of different treatments depending on the patients condition and labs, has not been invited. It is a travesty when such a clinician that could give so much to this meeting, is not invited. If I were you I would restrict your inflammatory comments-they only serve to divide the CFS community.

  5. karen says:

    I registered to attend the meeting. i did not register to make comments. Should I amend this? How would I do that?

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