NIH P2P Presentation by Susan Maier at IOM Open Meeting 1/27/2014

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Susan Maier IOM Open Meeting 1/27/2014

(Transcript by Patricia Carter)


Thank you. Thank you. Um

I have not been doing ME/CFS for very long. When I started at NIH with this, I knew absolutely nothing. And it took me a while to learn, and now I am very happy to be here today and to be involved previously and to continue my involvement with looking at ME/CFS…um… So, thank you for letting me come and talk to you today. I appreciate the invitation and I hope you will get out of this what you need to know and of course you can ask me questions after. So. Let me get the right direction here.

Let me first start by telling you that the Pathways to Prevention Program is a collaboration. This is a collaboration between the National Institutes of Health, Office of Disease Prevention, and the Trans-NIH ME/CFS Research Working Group. They are the ones who submitted the proposal to the ODP (Office of Disease Prevention) for the Pathways to Prevention Program. As you can see from the slide, the goals of the program are fairly straightforward: Identify research gaps and methodology problems, identify weaknesses in a scientific area, suggest research needs, and move the field forward through unbiased and evidence-based assessments of a complex issue. Complex health issue. Complex public health issue.

The Office of Disease Prevention does all of this with a very, very high level of coordination. They provie infrastructure, they provide funding, and they provide leadership to the staff at NIH who submit these proposals and review them and …uh… then approve them to move on.

So the…uh… the Pathways to Prevention Program really is a program for any disease, any illness, any syndrome—anything that needs…uh…work, that needs an understanding, that needs a more in-depth… uh… understanding of where the field is at. Remember NIH is a research organization. We do research. Our whole goal is research. All right. It’s research that will eventually move…into, hopefully, translation and clinical care. It has a projection to do that. That’s why we do it. But really, we serve… uh… the community of researchers in order to make sure that the research is done well.

So the Pathways to Prevention Program applies to any illness. Myalgic Encephalomyelitis/Chronic Fatigue Syndrome was chosen. It’s a competitive process, and we were chosen to move forward.

The Timeline that you see here applies—again—to all diseases, all conditions, all syndromes—that are being considered under the Pathways to Prevention Program. So the Timeline’s similar, all right, and it’s very straightforward. I’m going to go through each one of these spaces of the timeline, so don’t worry if you don’t see it all here. Just to give you an idea of what our dates are like…okay, where are we…on this pathway? Here we go.

Okay, so if you look on the very far left, we started this process in June of 2012. And we’ve been going at this for a long time. But we started this process in June of 2012 when we first met with the Office of Disease Prevention, and we decided, hey, you know, there’s a real problem here. We need to do something. Um. The ODP received our proposal in November, they made a decision to accept it in December of 2012. We had an organization meeting in February of 2013, a year ago now, then the dark day in October of 2013 when we were supposed to have our first Working Group meeting to plan the Workshop, and what happened but we were all staying at home. All federal employees stayed at home. In my opinion, it was a dark day, but we got over it. Now, what that did, though, was it set us back in time. Okay. So we couldn’t have our Working Group meeting that plans the Workshop until January of 2014. So we just had it earlier this month. All right. And you’ll see the “tentative” okay? Let me repeat, “tentative” date for the Workshop is December of 2014. That’s what we’re gearing up for and that’s where we hope to be eventually.

So, again, here are these dates just to give you and idea… um… we meet to discuss what was called the Evvidence-based Methodology Workshop, now referred to as “Pathways to Prevention” Work…”Pathways to Prevention” Program, essentially the same program, it has the same goals, it has the same timelines, it’s just now called “Pathways to Prevention,” and I will continue now, from now on, to refer to it as “Pathways to Prevention.”

The Working Group submits a proposal, the proposal gets approved, the organizational meeting is the place where federal partners get together, and they review the proposal for merit. Should it move forward?

So, February 2013, picture this: We’re all sitting in a room. These are federal partners. Okay. The Working Group, the Trans-NIH ME/CFS Working Group, there’s the NIH representative, the Agency for Healthcare Research Quality was there. Office of the Assistant Secretary for Health was there… uh… the US Food and Drug Administration had a representative there, and other federal partners. These are federal partners who have an interest in investment in ME/CFS research. Many of them also sit on the Chronic Fatigue Syndrome Advisory Committee, and many of them are involved in the background with ME/CFS, especially the Working Group members at NIH who don’t come out to stand in front of you to talk about what they do. They don’t tell you how much and how hard they work to make sure that the research in this area gets completed, gets funded, is overseen and the results get published.

So the federal partners are asked at this organizational meeting—make the case why ME/CFS? Why now? And what effect will this workshop process have on the research moving forward.

It wasn’t hard to make the case. Okay. Necessity. Affected individuals have something wrong with them. It doesn’t matter what you call it, but they have something wro… there’s a common theme there. There’s a lack of diagnostic indicators. There’s a lack of understanding about the scope and the range, a lack of physician recognition of the problem. Okay. There’s an urgency. There is no FDA-approved treatment. There’s no cure. There are very, very few drugs in the pipeline in development, and there are very few drugs that are in trial phase. The current interventions that are used do not treat the illness. They address the symptoms. They’re not heading towards a cure. This is a public health issue. Okay. People are sick. There’s an unmet medical need. There was not any problem at all making the case to move this forward.

So the next phase of this is the Working Group Meeting. What the Working Group does in this process, the Working Group is composed of experts, content experts in ME/CFS, some federal partners, patient/patient advocates, caregivers, researchers, clinicians—people who have an understanding of the illness. They’ve been involved in the illness for a long time. Whether they have it, whether they do research on it or whether they are a clinician who provides care. There were three major tasks associated with this meeting, and believe me, the Office of Disease Prevention ran the meeting, and they kept us on task, on time. We had a very, very productive meeting.

So we had three tasks:

  • Refine the questions for the evidence review. The evidence review is performed by an Evidence-based Practice Center. Evidence-based Practice Center is a contract, and that contract is done through AHRQ (Agency for Healthcare Research Quality). It’s done through AHRQ, okay.

  • We had to develop the Workshop agenda: topics, speakers, format. All agendas—topics, speakers, format.

  • And we had to nominate panel members. I’ll get to this in a minute.

All right. So. We had to refine the questions. Here I’ve listed in bullet points just the five major, major categories of questions. I do have some breakout of those, and when I’m finished with this, I can go over that breakout so you get a better idea of what we mean, but essentially,

  • How do ME and CFS differ? Do they differ?

  • What tools and measurements help define subsets in what could be a spectrum of ME/CFS?

  • What are the characteristics of individuals who respond to certain treatments in the spectrum of the disorder?

  • What does research on ME/CFS tell us about the clinical diagnosis of ME/CFS? How is it related?

  • And have previous research findings really shaped clinical practice, or are research and clinical practice completely separate with respect to this illness?

So, again, these are just the basic questions that we started with. We had to take those questions and refine them. We did refine them down. The reason we have to refine them because the questions form the basis of the agenda for the Workshop. So, topics, speakers and format for a Workshop Agenda. The topics for the Agenda are defined by the questions that I just articulated to you. And how they are refined by that Working Group membership.

The speakers who will come to the meeting to talk about those questions are content experts. Okay?

Clinicians. Research… researchers. Those who have expertise in the diagnosis and treatment or conducting clinical and experimental research on ME/CFS. Those are the speakers. The speakers who will come there, address the questions, and who will provide an overview to the panel members. These are people who have an in-depth understanding of the illness. They represent a specific position…um… they could represent a pro or con position, so I think this definition is best. I don’t think this definition is very good at all. But we have balance… okay? We have a balance at that workshop so we have input from all sides. And they are recognized leaders in their field. They’re recognized as opinion leaders. They’re recognized as content experts in ME/CFS.

Now the Evidence-based Practice Center which is the contract who does the evidence review—they will also speak to how the evidence review was done. So there are specific ways and this is a confirmed methodology, for taking the evidence that is provided in the literature and honing that down to present or to articulate in summary findings from the clinical research. So each agenda session, which is a question or a topic area, will be followed by a Town Hall-like question and answer. All right? So people will have the option and the ability to, at the meeting, after each session is finished, to discuss, talk and have conversations about the presentations.

Of course, the format we’re kind of limited to no more than two days. This means if talks are no more than 20 minutes, do the math, we have enough content for a five-day meeting. We have to cram it into two days. And we’re trying to do that by refining the questions.

And of course, time for breaks and lunch, and of course, federal meeting, no food for feds, no food for feds who come to a meeting. It’s a fed meeting, so everything’s always on your own. The third task at that meeting was to nominate panel members. Well, who’s a panel member? What do you mean by a panel member? A panel member is a US citizen. Okay? They are highly recognized experts in their specific professional or public area. Okay? Highly recognized experts. They are not subject matter experts in ME/CFS. All right. They may be methodologists. They may be ethicists. Attorneys. Immunologists. Clinicians. Researchers. Um… health disparities folks. Public representatives. There’s a whole bunch of people who can be part of the panel, as long as they are not conflicted with the topic of ME/CFS. And of course we can’t have panel members who are agency or employees of the federal government. But just to give you an idea, a comparison, the workshop has content experts. The content experts serve to define the content area for the evidence review. They may be a speaker of the Workshop. They may be a member of that Working Group Committee. They may be a peer review or technical expert panel reviewer. Those are people that would all have a content expert perspective on ME/CFS. They can be conflicted. They should disclose that, but they may have an opinion, a bias, or they may hae some pro or con position on ME/CFS.

A Workshop Panel is just independent of ME/CFS, the topic in this case. They don’t have research, they don’t have publications on that specific topic, they’re not a federal employee, and they cannot be conflicted financially or research or clinic. All right.

So what happens next?

Well, there are three boxes on the slide. The one on my left is one process. The one on the right is another process. They occur at the same time. Okay? The one on the left, the ODP, the Office of Disease Prevention, who is coordinating this entire thing, they have all the content from the Working Group meeting we just held, they will set the date, they will engage the speakers for interest and availability and they’ll submit the conference approval form, and they will do a number of things related to the actual Workshop.

On the other side, we have the Evidence-based Practice Center who will start the evidence review. They will take the key questions that we send them, they will cull them down, and they will pull out, using the standard methodology, what types of research they will look for and what types of things they will pull from the literature to form part of that evidence review. All right? And they will create the evidence report. All right.

Now, one thing I have avoided telling you about because it’s kind of awful, is that due to efficient spending policies in the federal government, we have to have every single meeting approved. All right? Over a certain dollar amount. Even under a certain dollar amount. We want to get together and have a coffee… okay, not quite that bad but it’s pretty bad, all right? So this Workshop counts as a conference. It counts as a meeting. So we have to get it approved. Technically, according to the federal government, this Workshop does not exist yet. So, that’s why there’s a big blue arrow that says “approval.” We must obtain approval for the conference before we can move forward. Once we obtain that approval, we release the Workshop agenda, the speakers who will be coming to the meeting to speak on the topic, the logistics for the meeting, anything that anyone ever wanted to know about this meeting will be public. We’ll release the evidence report to the panel members and to the public, and we’ll widely promote the meeting. All right.

So. In summary, if you didn’t get that all with all the slides, all right. The Pathways to Prevention Program is a multi-step process involving a very high level of coordination. Topics are submitted to the Office of Disease Prevention selected by an independent panel at the Office of Disease Prevention based on research need. Different individuals with interest and investment are involved in developing the topic, the key questions, engaging experts, and creating the foundation content for the Workshop.

Foundation content consists of an evidence report and it’s based on key questions that are relevant to the field and the presentations from the content experts.

An independent panel of individuals who are recognized experts in their own field will assess the weight of the evidence from the report and from the speakers, what they present at the meeting.

The deliverable from this Workshop is a set of recommendations from the panel based on the totality of the evidence that they have read and heard. And a follow-up is conducted within one to nine months that then works on how to process the recommendations and move them forward into practice.

I want to encourage you to reach out to these people. Paris Watson is the person who is in charge of the Pathways to Prevention Program. She was glad to answer your questions. When she can’t answer them, she will forward them to someone else. You may reach out to me. I’m Susan Maier. And you may reach out to Dr. Mariela Shirley. Dr. Shirley is the new Chair of the Trans-NIH Myalgic Encephalomyelitis/Chronic Fatigue Syndrome Research Working Group. She is also the new ex-officio member representing NIH at the Chronic Fatigue syndrome Advisory Committee.


Thank you so much for your presentation. I do. I notice people taking pictures of slides. We actually will be posting these presentations on the website, so… I mean, feel free to do that, but they will also be available to you later. Um. Ted and then Lily.



Thank you, Susan.

I have one or two… depends on your first response


I’d better do it right…


The … At the end, there are some recommendations.




The NIH, you emphasized, was a research unit that hopefully would have products that would translate into practice, but are those recommendations, then, or research agenda for the NIH or is it recommendations for practice?


It has the potential to be both, but understanding that we are a research organization, and our focus is to improve the … um… integrity of the science that is used for translation into clinical care means that we have to focus on besting the science that is used for the evidence, so that is really our primary focus. But I have no idea what the… the panel will recommend.


As I was talking, I realized that it didn’t matter what you were to say, there was going to be two. Um… uh… You had your panelists and part of the sub-group was the clinicians, and in the group of clinicians there was nobody from primary care. And given that most patients with this disease, whatever we call it, are going to be seen in primary care, I think that adding primary care to the panel would be important.



Absolutely. Absolutely. This is just a… a smattering of the examples, and I… your suggestion is well… is well taken.


Lily and then Nancy.


I have three questions. So, the first question is, on the first task on the list of refining the questions, it says “What does research on ME/CFS tell us about clinical diagnosis of ME/CFS? That seems very similar to what the charge, at least what my understanding is, of what the IOM is being charged with. So, I wanted to understand a little bit about how that would work between the NIH and IOM. And then my other questions was, I’ve been reviewing the Pathways to Prevention Website to try to understand how the process works, and let me see if I have this correct.




So the panel, you cannot be a clinician that has ME/CFS experience and you cannot… or you cannot be a researcher that has published on it? So, that was concerning to me because this is a complex illness. We’ve talked about it—about that—before in a lot of different venues, and it seems like the P2P … I can understand its… the… goal lists out which topics it chooses, but one of the requirements for the topics was that it be non-controversial. And this is a field that’s undergoing a lot of changes. So I’m concerned that there isn’t going to be anyone that has significant clinical or research experience on that panel.

And then my third question is, according to my understanding, the panel writes the document in 24 hours after the workshop. So, that’s what I wanted to get an answer about.


Okay. Let’s start with the third one. Yes. So. The panel, after… they will receive the evidence report and they will receive any other documentation that’s submitted six weeks before the workshop. So they will have that content before the workshop and they are charged with reviewing it. At the Workshop, now remember, the evidence report covers a certain amount of research. It’ll be clinical trials, it’ll be clinical research. It won’t be any specific… it will be the evidence report. It’s a listing and a review of the evidence. At the Workshop, the panel will listen to the speakers, the speakers may address the content of the evidence report, they may add different information in there. They will provide perspective from the questions assigned as…their, you know, you’re expert in this, but they will also provide additional information that may not be in there. Give you an example. The CDC study may not be published yet. It might not be included in there; however, it would be very important for the panelists to hear about that study and what it hopes to accomplish. So, there will be that opportunity to split it up, and have the panelists listen to that. Yes, they write it within 24 hours. Within 24 hours of the Workshop, the recommendations are complete. They are working during the Workshop, so every evening, they go back to their little sequestered place. It’s a small “S,” not a big “S.” Um. They go back to their sequester place and they start writing. Okay. So they do have a day and a half. Total. Um. That report—the draft recommendations—will be posted on the ODP website for public comment, and for anyone to comment on. From there, it will come down after 14 days. All the comments will be reviewed, and the report will incorporate that content, and then it will be sent back out to the panelists for their review. Okay, that’s the one.

Um. The… I’m thinking here… yeah. There is a link between what the IOM is doing and what we are doing. There’s no doubt that there’s a link there. And so we hope to be able to work with the IOM Committee and our committee, or our Working Group, or whatever process we’re in at the time, to be able to share that synergy. There… there’s no doubt that there’s overlap in that sense.

I’m sorry. I can’t remember your third one. Was there a third question in there?


Yes, the question was, uh, if the panel requirements seem to imply that you can’t have clinical or significant research experience…


So the requirement of a panelist is that they may not be a clinician who sees patients with ME/CFS. They may not be a researcher who performs research on ME/CFS. They could be a clinician who treats pain disorders. They could be an oncologist who treats cancer-related fatigue. They could be an expert in a research area that has recently undergone a case definition or case definition review, such as a polycystic ovary syndrome. So they can… they are highly regarded experts in their field. They just are not ME/CFS experts. That content comes from the speakers at the workshop. That’s where those people will make their case. Think about this. It’s a jury model. Okay? You have the defense. You have a prosecution. They both know the case really well. They know the details, they know what’s going on, they know all the nuance, they know what’s going on in the media. Your jury is sequestered. They don’t know. They don’t know anything.

“I’ve heard of that, but I’m not sure. I don’t know about that trial." The jury hears the evidence and they make their decisions based on the evidence. That’s essentially what the panel is—the difference between the workshop speakers, the evidence report and the panelists—get it.


Nancy and then Cynthia.


I’m just so challenged because, you know, I’m on… both groups. My understanding, if I can paraphrase, is that IOM is not able to put anything out until its final report, so it would be very difficult to synergize, per se. But it can certainly hear. So it’s a one-way direction from P2P


Yes, it is.




I think we talked about that…


It sounds very important because… and… there’s so much that would be, you know, duplicated—literature reviews and so on. You have this extraordinary group doing a comprehensive review of the entire literature. Obviously, that’s something this committee could really value and not have to reproduce, that would be fabulous.

The case definition charge itself is… is difficult. If… if this group is really focused on a clinical case definition, um, and the P2P is really focused primarily on—in its case definition focus—that piece of it—research case definition, um, I think. At least that’s what I understand.


The goal of the P2P is not to develop a research case definition. But to review it. Review the evidence in supporting all case definitions that have been used.


It’s going to be very… this is very interesting.

So, anyway, how do you see the one-way direction of this information flow working? And I’m worried about the timelines because… when will the… when will the… working group have the…uh…the evidence …


The evidence review will be completed roughly six weeks before the workshop. We’re planning for December 2014. Planning. “Tentative,” please. Planning.

So, six weeks minus will be when the evidence review should be completed and should be sent, then, to the panelists.


Might it be possible, then, in order to facilitate this group’s work, which synergizes, to ask that the case definition part of that be up front and available to this committee sooner than that because our timeline, I think that’s too late… to be useful.


Yeah. I think the IOM timeline is shorter. Yes. Um. I… I… I… don’t have a thorough answer to your question, but let me tell you th…Let me tell you what I know.

Um. The two processes are different. And our—not a charge—but our purpose is different, and so the questions that we develop will be heavily focused on the research part of it. That doesn’t mean that the research part of it…it will not be informative to a clinical case definition, but it means that our questions and, therefore, our evidence review, might not exactly dovetail with what you’re looking for. That doesn’t mean it isn’t … uh… usable content. It just means that it… because what we’re reviewing is different than what you’re going to review, as the IOM.


So, can I just ask in a simpler way of asking then. Do those of us who are on both committees —I think there are two of us here that are—uh, permission to share what we have to date. Because we were given interesting content just two weeks ago.




That was very good. But I don’t know what my limits are in sharing that—with this committee.


I think we discussed at that meeting that it would be because, well, a…

Everyone as you may…as…as the people who were at the meeting may well know, um, the ODP coordinator went off on vacation for two weeks. (laughs) She just got back today. Um. She does a fabulous job, but she’s got to get back to what she was working on. So the content of the meeting is not finalized. All the things that we discussed are on…in …you know… sheets of paper and maybe a couple of computer disks, okay, I won’t say diskette, but a couple of things here and there. She has to pull that all together. When she pulls it together, there may be some semblance of order that she can say, hey, this was what I’m going to distribute to the Working Group members. At that point, then you can share it. Because it would be, okay, well here’s some content that I have that I have knowledge of that I might find informative for other committees on which I sit.


I’m totally confused.


Are you?


I’m just thinking that I have a folder, you know, a lovely two pages of paper with a beautiful case definition chart with all the case definitions.




Revolution seems like a perfect document to start some conversation here, but I don’t know my limits in sharing that little piece of paper.


I think that Paris Watson at ODP is the one to give you that specific guidance, just to be sure.


My understanding is that protocols for evidence reports are supposed to be publicly available.




So, uh, we should be able to have access to the protocol for that evidence report. I am wondering if there are ways that we can work such that we get access to the search strategies? That are used in that evidence report, tied to specific questions that are addressed that presumably are already specified in the protocol so that we can use those before the final report comes out? And also, my understanding is that these evidence centers develop evidence tables before they have finalized the report and I’m wondering if a way could be worked out so that’s an interim product, really? If there could be a way worked out such that our committee could have access to some of these interim products?


I think those are very valid questions, and I apologize. I don’t know the answer to that at this point in time, but I will certainly go back and ask. I’ll find out what the Statement of Work says about the work products deliverables, when they’re due, who they get released to, etc. But that’s a very valid question.


Well, thank you very much. I really appreciate this.

Uh. I think what we ought to do now. I know that we’re not scheduled for a break until three o’clock, but I would actually rather take it now. Uh. Uh, in part because … well, mostly because that will give us some more time when we come back to hear… to hear from the patient perspectives. For those of you who haven’t noticed, there have been little signs going up here when people get near the end of their time. There’s like a yellow sign and then a red sign. So, … and we will be doing that afterwards for the many talks that we’re getting ready to have but with your permission what I would like to do is take the break now, reconvene at ten till three, for real. And then start at the … start with the voice of the patient presentation and then start hearing from our various …um… from our various representatives. So, with your permission I would like to adjourn now for 15 minutes. So we’ll reconvene when that clock says ten till three. It’s late. So don’t … yeah, I know.

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