FDA Progress on Measuring Outcomes

I have an update on the progress of the ME/CFS Outcomes Measures Working Group working with FDA.

As I reported in March 2015, the FDA helped convene a Working Group comprised of representatives from FDA, NIH, CDC, and academia in order to create and qualify a clinical outcomes assessment tool for ME/CFS. This is critically important in the drug development process. If FDA officially qualified an assessment tool, then it could encourage drug developers to get into the ME/CFS area. Such a tool does two things for drug developers: a) it saves the money of creating and qualifying the tool and b) they know that FDA will accept use of that tool in clinical trials.

I am happy to report that our effort to create the tool has officially been accepted into FDA’s Clinical Outcome Assessment Qualification Program. This means that FDA has reviewed and provided guidance on the design of a study to create the tool. The Working Group plans to test whether items from NIH’s PROMIS question bank are applicable in ME/CFS.

The Working Group understands that this is not simply a matter of measuring fatigue. In fact, figuring out how to measure post-exertional malaise is one of the puzzles we are tackling. It may turn out that PROMIS items don’t work for the ME/CFS population, but we need to test that. Starting from scratch to create a new measure takes much longer and is more expensive.

Right now, we are actively seeking funding for the study. I’ll keep you posted as things develop.

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16 Responses to FDA Progress on Measuring Outcomes

  1. cort says:

    Great news Jennie! Thanks for sharing…

    Where is the group looking for funding from? The NIH might/should jump on this progress in this critical part of the field…

    • Jennie Spotila says:

      NIH is definitely one potential target. Other proposals are in the works.

    • Cort says:

      Am I right in that you’ve found a tool that you think works and the tool is located in Promis?

      • Jennie Spotila says:

        No, the study will test the hypothesis that PROMIS items will work in this population. We don’t know if they will.

        • Cort says:

          Can you say who’s in the Working Group? Is Lenny Jason, for instance, in there?

          • Jennie Spotila says:

            I don’t have clearance to list the group members. I’ll check on whether I can release names. I do think it’s fair to say the Working Group has spoken to multiple collaborators in addition to the Group membership.

  2. Sarika ( Sara) says:

    Thank you for the updates!

  3. Leona says:


    Thank you for all your hard work. This, plus the announcement by NIH, gives me real hope that answers may be found.


  4. Adrienne says:

    In my opinion a tool for measuring ME/CFS could be a comparison of resting heart rate and heart rate variability in the 4 days after an “exercise” challenge. I find after too much exertion, my resting heart rate increases markedly the next day, my HRV drops. It takes 4 days of aggressive bed rest to get back to my baseline. The pattern is so consistent. For me if my true resting heart rate is up 10 bpm the day after doing something, I know that I have a 4 days of total flat bed rest to reduce my symptom load and true resting heart rate back to baseline.
    I think we have objective tools to measure the impact of exertion especially in the case of bedridden/housebound people who don’t have the same volume of extraneous inputs into their days.

    • Jennie Spotila says:

      I agree there are several candidate objective measures. Those go through a separate FDA qualification process. The idea behind this specific project is to use PROMIS measures because those are so well qualified and accepted in other clinical settings. But this does not preclude someone from entering an objective measure into the process. Last time I checked, there were no applications for objective measures or biomarkers in ME/CFS, although perhaps that has changed.

  5. janine says:

    Hi Jenny,

    I would like to see a ‘fast track’ process where existing FDA approved drugs could be repurposed to treat ME/CFS. Do you know if this is something the FDA is currently investigating?

    Thank You!

    • Jennie Spotila says:

      Good question, Janine. FDA does not conduct that kind of testing itself. Instead, a drug developer would have to file an application to have its drug approved for ME/CFS. FDA does have fast track review programs but the application has to be initiated by the developer. So for example, the developer of Rituximab would have to file an application to have the drug approved for ME/CFS. I am not aware of any applications pending like that. Certainly having an outcomes measure qualified by FDA might encourage this kind of an application, though.

      • janine says:

        Hi Jenny, Thank you for staying on top of the progress, and keeping us informed!!

        Why isn’t the 2 day CPET test adopted as a measurement tool to diagnose and track the progress of PWME? I consider the CPET test to be the ‘gold standard’ since PEM is the qualifier for ME. It’s an easily repeatable test that uses scientific measures. I realize some people are too sick to take the test, and other measurement tools will need to be considered.

        • Jennie Spotila says:

          Two day CPET could be proposed as an endpoint in a drug trial. In fact, the Ampligen trials used an exercise test (but not two day CPET, if I recall correctly).

          This effort of the Working Group to create a clinical outcome measure does not preclude any drug developer from using a biomarker as an endpoint. However, the drug developer has to prove the biomarker is valid and useful in the specific context. In other words, if a drug changes NK cell count, the developer has to demonstrate that changing NK cell count makes people with ME/CFS more functional.

          There is a separate process for biomarker qualification. A researcher could submit an application to qualify two day CPET or NK cell function as a biomarker for ME/CFS drug trials tomorrow. To my knowledge no one has yet.

          Clinical outcomes measures and biomarkers are not mutually exclusive. Nor is it my impression that FDA is not interested in ME/CFS biomarkers. We’re trying a short cut to get a measure qualified. But I feel very strongly that biomarkers will serve us better in the long run and I hope researchers will enter the process to qualify them for use in clinical trials.

    • Beca says:

      Stellar work, Jennie. Janine, great request, and let’s invite everyone to submit their successful orphan drugs and supplements to the right group/s. The mysterious and sudden turnaround in funding for and interest in NEID is, let’s intend, a harbinger of a huge apology backed by even greater funding, acknowledgement and cure of this monster condition by the powers that are gaining strong power: Us.

  6. rivka says:

    thank you for all this hard work. i can’t comprehend it all right now, my brain won’t allow that. so i’m thrilled that you and others can. — xxoo rivka

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