Staying the Course to Where?

which wayDr. Francis Collins, Director of the National Institutes of Health, made a request of the ME/CFS community. During the March 8, 2016 NIH telebriefing, Dr. Collins said:

So please take our commitment with great seriousness. Please also stay the course with us as we seek to identify the most compelling research questions and how we could address those. (emphasis added)

But what precisely is the course Dr. Collins has asked us to stay on? We cannot answer that question yet, but we can begin to sketch out the map. We can also identify the elements we expect. Looking across the various communications from NIH to the public in the last several months, here is the outline I see:

Clinical Care Study

The NIH has already designed and has begun executing a study at its Clinical Care Center. There are so many issues and questions about this study, including the risk of bias, the design of the exercise test, patient selection, and more. I refer you to the webinar with Dr. Avi Nath, the principal investigator, in which he addresses some of these issues.

I hope to cover the study in more detail in a future post. But one thing to keep in mind is how long this study will take. Data collection for phase one will take approximately two years. Analysis and writing up results will take longer. And there are two additional phases after that. I don’t think we’ll see even preliminary data until the end of 2017, at the earliest.

Short term plan to activate research

Dr. Vicky Whittemore made general references to a short-term plan for research on the NIH telebriefing. She said, “We are in the process of putting together . . .  both a short-term plan, where we can try to activate some research on the shorter-term as well as initiative that would put in place better infrastructure as well as research funding for longer-term research projects.” Dr. Whittemore also said that the research plan would be presented “to the appropriate council for approval in the May time frame.”

The specifics of what this plan will include is anybody’s guess. Dr. Whittemore did note that the Trans-NIH ME/CFS Working Group has been discussing the importance of biomarkers, understanding the underlying causes of ME/CFS, and understanding the cognitive dysfunction associated with the disease. But there are so many more areas that need investigation, including characterization and evaluation of post-exertional malaise, investigating the possible autoimmune connection, expanding the systems analysis of neurological and immunological gene expression, genome wide association studies, and clinical trials. In fact, the P2P report includes a long list of areas of inquiry.

It is also important to note that this plan appears to have been put together by NIH alone. It is unclear if NIH has consulted anyone outside the Institutes for advice and direction. And there has certainly been no systematic collection of public input (although advocates have been offering their thoughts). We also don’t know if the plan will be released in its entirety, or if announcements will be limited to specific initiatives. The last time the Trans-NIH Working Group created any kind of plan was after the State of the Knowledge meeting in 2011, and NIH steadfastly refused all requests to see that plan.

Requests For Applications (RFAs)

RFAs are a big deal to us because they set aside funds for particular projects or areas of inquiry. The last RFA for ME/CFS was 10 years ago. Advocates and researchers have been clamoring for another ever since. It seems like we will, at last, get our wish.

Dr. Walter Koroshetz would not commit to RFAs on the telebriefing, but he said, “I think as a short-term process that we definitely have to stimulate with funds that are particularly for ME/CFS and Dr. Collins is clearly behind that.” Afterwards, NIH issued revised responses to CFSAC’s August 2015 recommendations, stating, “The Trans-NIH ME/CFS Working Group is in the final stages of putting together a comprehensive research strategy for ME/CFS research that will include new RFAs.” (emphasis added)

This is good news! But it is nonspecific news. We will have to wait for the announcement to find out how much money will be allocated and over what period of time.

Long term plan with coordination

It’s a bit fuzzier trying to map out what will happen over the long-term. Dr. Koroshetz said, “But I would also emphasize once again that this is a stepping stone. . . . what our intention is, is to coordinate it with many of the other pieces that we’re going to start initiating across the country.”

He talked about organizing researchers around the country, and that the community should work with investigators at universities and clinics. Such groups would then submit funding applications. I think this could take many forms, and not all would be equally helpful.

Common Data Elements and a Data Coordinating Center

In the revised responses to CFSAC, NIH said it will develop Common Data Elements for use in all studies. NIH also said the Trans-NIH Working Group is exploring the feasibility of a Data Coordinating Center. This represents a change from NIH’s previous position. In response to CFSAC’s recommendations from June 2014, NIH said “developing and maintaining a unique ME/CFS database is cost prohibitive in light of the small number of ME/CFS researchers.”

CDEs and a central data repository are much needed, especially given that ME/CFS specialists and researchers are scattered across the country with limited systematization of data collection. Again, we await specifics.

stay the courseMy Roadmap

In order to decide if we will “stay the course” with NIH, we should have a sense of what we think that course should look like. These are the components of a course that I would be glad to stay on with NIH:

First, NIH should share its research plan with the public. Not only does this increase transparency and accountability, but it will be another small signal that NIH wants to improve its relationship with the ME/CFS advocacy and research community.

Second, NIH should begin with a three year sequence of RFAs. Starting with $10 million this year, and ending with $20 million in 2018, the RFAs should support validation of biomarkers, early clinical trials, and infrastructure. NIH should then evaluate results to appropriately target future RFAs.

A sequence of RFAs is essential. Our last RFA was one and done, and it has taken 10 years to get NIH to agree to do it again. We cannot wait 10 years between RFAs. Furthermore, dedicated funding over several years is necessary to convince researchers that NIH is serious about its focus on ME/CFS and to attract new researchers to the field. As long as the perception lingers that NIH does not really want to invest in this disease, we will not see research move forward with any increased speed.

Finally – and this might be obvious but should be explicitly stated – we need the first RFA to be this year. NIH cannot wait for the completion of phase one of the Clinical Care study to shape RFAs. That means waiting two years or more, and it is completely unacceptable.

Third, NIH must fund Centers of Excellence. This might be what Dr. Koroshetz was referring to when he talked about coordinating efforts across the country. NIH should help recruit three major universities within two years to combine ME/CFS expert clinical care, research, and clinical trials under one roof. These Centers should also participate in the creation of Common Data Elements and the Data Coordinating Center. We need NIH’s support and a plan of how we will get these Centers up and running.

Fourth, NIH must incorporate public input in planning and executing ME/CFS research initiatives. Dr. Nath said during his webinar that the “extramural folks” were “approaching people and putting together a panel.” I confirmed with Dr. Whittemore that they are working on this, but she had no further information to release at this time. It is absolutely critical that NIH consider the range of views among patients, advocates, clinicians and researchers – and meaningfully incorporate that input into designing RFAs, studies, data elements, and future initiatives.

Fifth, NIH must bring stakeholders together to agree upon a basic research case definition, that can then be refined for the needs of particular studies. This is what the P2P Panel recommended, but case definition has been completely absent from NIH communications thus far. NIH says it wants to recruit new researchers to this area, but those researchers need direction on which disease is the target. We need to put a stake in the ground on this issue and move forward.

Finally – and I realize this is not the nature of government – we need a sense of urgency. NIH announced its renewed focus on ME/CFS in October 2015. We’re hoping to hear a plan in May 2016 (although no announcement date has been set). The Clinical Care Center study will not yield data for several years. RFAs take time to produce grants, and those grants take time to produce results. I want to see the NIH move much more quickly and with a greater recognition of the urgent need for progress. We’ve already waited thirty years. That’s long enough.

This entry was posted in Advocacy, Commentary, Research and tagged , , , , , , , , , , , , , , , , , , , . Bookmark the permalink.

17 Responses to Staying the Course to Where?

  1. Sharon Kramer says:

    You are doing some great work of watch dogging the gov’t backed ME/CFS research.

    FYI, There is an exciting emerging field of research that I believe is highly relevant.
    Check out this new post from the University of Virginia, “They’ll have to rewrite the textbooks”

    “Now we can approach this mechanistically — because the brain is like every other tissue connected to the peripheral immune system through meningeal lymphatic vessels,” Kipnis said. “We believe that for every neurological disease that has an immune component to it, these vessels may play a major role.” –

    See more at:

  2. I’m glad there are people like you keeping the NIH’s feet to the fire.

    Too bad they appear to be wearing fireproof boots.

    I can’t do anything about this – neither could most of the people who died of AIDS or Ebola or the plague.

    I hate that I depend – for my life – on my disease being ‘interesting’ to a bunch of medical researchers somewhere.

    That’s not how public money should be spent.

  3. jimells says:

    Two billion for Zika.
    Two support staffers for M.E.

    And people are still telling themselves that NIH wants to solve this puzzle.

  4. Chris says:

    Jennie, a great piece–calm in tone, absolutely clear and based on the kind of fully updated info that you can extract from them–we can only hope that “they” are reading and absorbing your points. But there is still some hope, I hope… And of course we have no realistic options to “staying the course.” They are in control, they hold the purse strings.

  5. Bobbi Ausubel says:

    Address for Collins. Who else to write to ar NIH.?
    Thanks for this.


    • Write your congressional delegation. Go to to find your two senators; go to to find your Representative.

      • The number one goal is for people to reach their senators and congress reps, but beyond this, anyone with time can also Tweet to anyone even outside district, 100 senators, 435 in house if they have time, since Tweets do not have zip codes or states attached.
        Key is to focus on reps on appropriations committees.
        Labor oversees HHS:
        Labor Subcommittee Members

        Tom Cole, Oklahoma, Chairman
        Mike Simpson, Idaho
        Steve Womack, Arkansas, Vice Chair
        Chuck Fleischmann, Tennessee
        Andy Harris, MD, Maryland
        Martha Roby, Alabama
        Charlie Dent, Pennsylvania
        Scott Rigell, Virginia

        Rosa DeLauro, Connecticut, Ranking Member
        Lucille Roybal-Allard, California
        Barbara Lee, California
        Chaka Fattah, Pennsylvania

  6. Magdalena says:

    Excellent as always, Jennie. Thank you so much for doing this. Much needed and warmly appreciated <3

  7. John Gabor says:

    The first priority should be research criteria. Without it, we and the researchers have no idea if they ate studying ME, or CFS, or just CF.

  8. Kathryn Stephens says:

    If NIH funded this, and NIAID has been working it for 3 years, what then, is NIH going to study…

  9. Well said.

    I wish they would also listen to us in our concern that the 5-member “expert committee” for the in-house ME/CFS study has the wrong expertise, and that with only 40 patients planned to be studied, we have reason to be concerned. GIGO.

  10. Rivka says:

    I am hearing more and more from patients who are now totally bedridden and can’t even walk to the bathroom. We need help URGENTLY. — Rivka

  11. Scott says:

    All great points you made Jennie. On your last point about a sense of urgency I wish we would also see that in the research community. Other than Dr. Ron Davis and OMI I fail to see that drive to solve this quickly. Yes, we need help urgently!

  12. kathy d. says:

    Thank you for your diligence on this. It’s appreciated.

    Well, there is enough red tape here to fit around the world twice. ME/CFS isn’t viewed as an emergency, as an epidemic that kills many people or renders babies with terrible birth defects.

    I just hope that the cause and treatment will be found in my lifetime — and I’ll be 70 soon, sick since 39 with this disease.

    I’m not counting my chicks until they hatch is my philosophy, so when I
    see concrete numbers in funding and more details, I’ll be more optimistic.

    I do not have a problem with funding for Zika or Ebola. And that was a
    fight with Congress, to get the Zika funding. Sufficient funds were not
    allotted and now the medical agencies are taking funding from that for
    other diseases.

    I say take the funds from the military budget; trillions go there and it’s
    for destruction, not the preservation of human health and life through
    medical research and treatment.

  13. Joe Landson says:

    This is surreal. Dr. Collins want to convince us his agency is serious. Almost none of his minions are serious, and they are proud to tell us so. Dr. Nath doesn’t want to find biomarkers, and doesn’t want to be an expert; he said so. Dr. Wallit doesn’t think we have a disease so much as what, an illness experience? We know because he said so. Dr. Kitt implied we don’t need extramural funding, because ‘good science always gets funded’. She said so.
    Dr. Whittemore seems the only exception, and she is sorely outnumbered.
    Dr. Collins really wants to convince us his agency is serious. Thing is… who’s gonna convince his agency that he’s serious? I don’t see anyone doing that. Not even him.

  14. billie moore says:

    A very nice analysis, as usual, Jennie. Skepticism is certainly in order when it comes to the NIH, or CDC. Joe Landon also nails it again.

    Why, however, ask for so small a sum as $10 million, up to $20 million in 2108? That will not fund much research at today’s research prices. We need the $250,000 we have been requesting, and if that is not feasible immediately, as it seems it is not, then we need to drop no lower than $100 million. I am not willing to stay a course of $10 million in additional funding – even $20, not only because it is far too little for the need of the burden of this disease, but because then we would be told, “Look what we did for you. (Go away.)” And if the NIH is not willing to bring in experts and patients to the process, there is no course to stay with.

Comments are closed.