Posts Tagged ‘funding’

Research Roadmap

April 14th, 2014 12 comments

Road MapThe Research Recruitment Working Group of the CFS Advisory Committee has been formulating recommendations that could potentially change the direction of ME/CFS research at NIH. Not much time has been spent on it at the last two meetings, but I think you need to pay attention to this. Dr. Dane Cook, chair of the Working Group, spoke with me about where they’re headed.

The Working Group was charged with two tasks: 1) increase awareness among researchers about ME/CFS research and 2) suggest strategies to increase the number of interested researchers who will apply for funding. Most advocates, myself included, have argued for the “build it and they will come” approach. If more money is made available for ME/CFS research, then more researchers will apply. Dr. Cook pointed out that CFSAC has been recommending increases in funding and RFAs for years without any success. In his opinion, it is time to try a different recommendation strategy.

Dr. Cook and the Working Group presented interim reports at the December 2013 and March 2014 CFSAC meetings. The Group has gathered data on the low number of CFS publications relative to the number of publications on both fatigue and fibromyalgia. They have also identified multiple barriers to increasing the number of interested researchers and retaining them in the field. I asked him to walk me through the three prongs of the Group’s current approach, with the caveat that this is not the final recommendation from the Working Group.

A Research Agenda Informed by the IOM and P2P Reports

The first step in the research road map is to articulate a clear research agenda based on the information and recommendations from the 2011 NIH State of the Knowledge meeting, as well as the forthcoming IOM and P2P reports. Combined, these three reports should identify gaps in the research and the priority areas for future inquiry. The IOM report may also resolve the dispute over the case definition, although it should be noted that IOM is creating a clinical case definition not a research definition.

Dr. Cook was pressed hard at the March 2014 meeting on the issue of urgency. The P2P report will be issued at the end of 2014, and the IOM report is not due until March 2015. The formulation of a clear research agenda wouldn’t begin until after that. Billie Moore and other CFSAC members expressed dismay at this timeline, and pushed for an immediate RFA. Meanwhile, a recent Congressional effort made a similar request of NIH, but this has come under fire from some advocates who believe that no money should be requested from NIH without guarantees of how it will be spent. They point to the recent denial of funding to Dr. Lipkin as proof that NIH cannot be trusted to make the right grant decisions.

Dr. Cook told me that the delay of waiting for the reports is the hardest issue for him personally. He would much rather see an increase in funding immediately. However, he pointed out that CFSAC has already pushed for this for many years. His assessment is that if CFSAC recommends another RFA now, the answer from HHS will be that they need to wait for the reports. Dr. Cook’s goal is to provide so much evidence of necessity that HHS will be compelled to act.

Championed by the Trans-NIH Working Group

The second prong of the road map is for the research agenda to be clearly communicated and championed by the Trans-NIH ME/CFS Working Group. Dr. Cook’s sense is that NIH is generally supportive of how he’s been working on this charge, but he did not articulate what “championing” would look like.

It’s important to remember that the Trans-NIH Working Group does not have a research budget, nor does it make the decisions on funding ME/CFS grants. But what it can do is bring people together from the NIH Institutes to promote ME/CFS research at NIH. Any step in that direction is a positive one, as long as the research is physiologically oriented and focused on the correct patient cohorts. Whether this could be achieved – and to what extent the Trans-NIH Working Group would evangelize it – is not entirely clear to me.

Strong Infrastructure

The final prong of the road map is to support ME/CFS research with a strong infrastructure. Dr. Cook is passionate about this, and believes that it could be undertaken immediately without waiting for the IOM and P2P reports. Currently, data sharing among ME/CFS researchers is piecemeal. Many researchers use REDCap to collect their data, and the system is designed to build and manage surveys and databases online. It’s an electronic data capturing system, not a system for aggregating and sharing data.

The National Database for Autism Research (NDAR) is a striking alternative model. NDAR was launched by NIH in 2006, and it offers both a data repository to facilitate data sharing and standardization, and a scientific community platform that offers access to other research repositories housed by other institutions. Applicants for NIH funding are strongly encouraged to contribute their data to NDAR, and data on almost 70,000 individuals with autism are available. Several NIH Institutes provide funding for NDAR, averaging about $2 million per year.

NDAR is far larger and more sophisticated than any ME/CFS data effort. Dr. Cook believes that ME/CFS research is in desperate need of such a resource. He also said that this could be pursued immediately, without waiting for the IOM and P2P reports. The big question is (as always) funding. An NDAR representative told me that the system could be rolled out for another disease area, such as ME/CFS, for about a quarter of the annual NDAR investment. But still, is NIH willing to invest $500,000 per year in building such a system for ME/CFS?

Where From Here

Dr. Cook indicated that the Working Group is continuing to refine its recommendation. His CFSAC term expires in early May, but he hopes to remain on the Working Group to continue and support the effort to finalize a recommendation to the Secretary.

I think many important questions remain: Is it appropriate to make the RFA contingent on the release of the P2P and IOM reports? Is such a delay acceptable? Who will be charged with articulating the research strategy? Will that person/group be willing and able to depart from the P2P and IOM recommendations if needed? Will the Trans-NIH Working Group champion this agenda and request an RFA? What does that look like? Who will be tasked with creating an NDAR-like infrastructure? Who will pay for it?

And the obvious question is: how long do ME/CFS stakeholders have to wait to see the investment of funding that this we so desperately need and deserve?


Congress: We Need An RFA

April 2nd, 2014 34 comments

I am very happy to report that an effort is underway to secure Congressional support for a $7-10 million RFA for ME/CFS funding at NIH. And there is something YOU can do to help!

Representative Zoe Lofgren (D-CA) and 10 of her colleagues have signed a letter to Dr. Francis Collins, Director of NIH, asking him to follow the recommendation of the CFS Advisory Committee and allocate $7 to 10 million for an RFA. This would be money set aside for ME/CFS research (currently no money is guaranteed to ME/CFS). I’ve posted a copy of the letter for you to read and take to your own Congressman/woman.

What you can do:

  • Read the letter, and if your Representative has already signed then call his/her office to say thank you! This is very important because these offices track the feedback they receive. So call your Congressman’s office, and say: “I (my family/friend/etc) am a constituent, and I want to thank the Congressman for his/her support of research into the medical condition myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS).”
  • Thank Dr. Ben Gutman, the aide in Congresswoman Lofgren’s office, for making this happen. Email him at ben.gutman AT
  • If your Representative has not signed the letter, then ask him/her to do so! Call the office, identify yourself as a constituent, and briefly tell them why ME/CFS research is important to you. Then ask that your Congressman/woman read the letter and consider signing. You can share both the letter and the introductory email (which begins “Dear Colleague”) with the office, because that email provides the context and contact information if they have questions. Do not worry if you only speak to a staff person and not your Representative. Congressional staffers are influential. Tell them that you will call back to follow up in 2-3 weeks – and then remember to do it.
  • Report results. If your Congressman/woman signs the letter, then please let me know. Just post the name, state and Congressional district here. And if he/she did not sign, politely ask why and report that reason and the Representative’s name here, too.

I’m not responsible for getting this ball rolling, but it’s nice to see. I’ll be calling my Congressman tomorrow, and I hope you will too.


2013 NIH Spending on CFS Studies

March 31st, 2014 26 comments

gold-pricesI have positive news to report: NIH spending on ME/CFS  in 2013 was actually higher than it was in 2012. Are you shocked? I know I was. NIH lists a total of $5.1 million for ME/CFS research in 2013, an increase of 13% over 2012. And for the first time ever, I think the numbers look better on closer examination because of how the spending was allocated.

The problem is not fixed, by any stretch of the imagination. ME/CFS spending fell to 226th out of 237 categories (we were 224th in 2012). Hay fever got almost twice as much funding; fibromyalgia got more than twice as much; TMJ got almost four times more; and multiple sclerosis received more than 22 times as much funding as ME/CFS.

I think it’s important to shape our advocacy based on evidence and facts, so let’s dig into the numbers. NIH had projected that it would spend $5 million on ME/CFS research in 2013 (see my previous analyses of of spending in 2011 and 2012). There are 16 grants listed for 2013 spending (one grant is listed twice because funding came from two institutes) for a total of $5,118,721. This is an increase of $600,539, or 13.3% from the 2012 funding.

Unrelated Grants

Last year, I found that 18% of the money NIH said it spent on ME/CFS was incorrectly categorized. This year, I am pleased to report that only 1.5% of the spending was unrelated to ME/CFS. The study by Dr. Matthew Hayes received $77,200 in funding to investigate the potential mechanisms that cause nausea and malaise after the administration of a class of drugs for diabetes. Just like last year, I still don’t understand why this is counted in the ME/CFS category, but the grant is scheduled to end in 2014 so hopefully this will be the last of it.

Category Breakdown

After deducting the unrelated study, we are left with total ME/CFS spending of $5,041,521. Let’s see the category breakdown:

When compared to previous years, the numbers look even better:

2009 2010 2011 2012 2013
Total spending $4,844,044 $6,194,042 $6,346,148 $4,518,182 $5,118,721
Not CFS Related 7% 6.5% 0 1.77% 1.5%
XMRV 15% 29.3% 27.5% 16.43% 0
Psychological 12% 12.3% 13.5% 20.14% 10.4%
Orthostatic intolerance 25% 13.5% 13.5% 7.01% 11.7%
Neuroendocrine Immune 42% 38.3% 45.5% 54.65% 76.4%

Look at those numbers! Psychological spending was HALF of what it was in 2012. That money, and the money spent on XMRV last year, has now moved over to the neuroendocrine immune category (including biomarker studies) to bring that category to its highest since at least 2008. This is a very good trend.

Several additional points of interest. First, the Office of the Director contributed $600,540 towards the studies by Dr. Jason and Dr. Shungu. The Office of the Director has provided funding in previous years, such Dr. Brigitte Huber’s study in 2011 and Dr. Natelson’s study in 2012. However, the 2013 contribution from the Office of the Director is far higher than in previous years. I’m not sure what accounts for that significant increase.

Second, there were four new grants in 2013 (just like 2012) totaling $1,763,585, or 34.5% of the overall total. This is an increase of $737,208 over 2012′s new grant spending. All four new grants were reviewed by the CFS Special Emphasis Panel, just like 2012. In fact, all of the external grants on ME/CFS were reviewed by the CFS Special Emphasis Panel.

Upward Trend

Perhaps the most important metric for NIH spending on ME/CFS is to compare the real numbers year by year. I’ve removed all the spending that was not related to ME/CFS (including XMRV in 2012), and here is the trend:

Adjusted Spending $ Increased (Decreased) % Increased (Decreased)
2008 $3,175,262
2009 $3,810,851 $635,589 20%
2010 $4,248,535 $437,684 11.5%
2011 $5,009,672 $761,137 18%
2012 $3,696,068 ($1,313,604) (26.2%)
2013 $5,041,521 $1,345,453 36.4%

In terms of real spending – i.e. money spent on grants actually related to ME/CFS – 2013 spending was the highest since 2008, and included the biggest increase (both $ and %) since 2008. I think this is a trend we could all get behind.

Prove It

While these numbers are good, the overall problem is not solved. Five million dollars is pocket change in scientific research, and grossly inadequate given the economic and human toll of ME/CFS. Dr. Ian Lipkin stated publicly that his application for a microbiome study was recently turned down by NIH, although we don’t know which review panel scored the grant or why it scored poorly. One source told me that the ME/CFS Special Emphasis Panel reviews approximately six applications each cycle, which means that applications have not increased in the last year. Multiple factors contribute to the low NIH funding for ME/CFS, and we will need multiple solutions to fix the problem.

Still, the funding for 2013 was higher than the funding in 2012, and I applaud NIH for that. The real question is whether this is a fluke, or the beginning of a trend. I would like nothing better than to report 36% (or more) increases for the next five years.


Exit Stage Right

March 13th, 2014 19 comments

Another CFS Advisory Committee member has resigned.

After the March 11, 2014 CFSAC meeting, I emailed the Office of Women’s Health and asked for a list of who had attended the meeting. I tried to keep track of the roll call, but there were clearly technical difficulties that prevented several members from answering and it wasn’t clear when they arrived. The CFSAC Support Team responded yesterday:

All of the voting members of the committee participated in the webinar yesterday (n=10), so a quorum was present.  (We have one vacancy – Dr. Dimitricoff [sic] resigned a few weeks ago.)  All voting members were present at the start of the webinar, except one member who was ~ 5-10 min. late. (emphasis added)

I have to wave several red flags here, and I would jump up and down too, if I could:

Red Flag #1: As of this post (March 13th), Dr. Dimitrakoff is still listed on the CFSAC roster.

Red Flag #2: No one mentioned Dr. Dimitrakoff during the March 11th meeting. I thought it was odd that Dr. Marshall didn’t call his name during roll, but assumed that he was simply unable to attend. It is completely inappropriate not to announce to the public that a member has resigned! Do the other CFSAC members even know? When were they told? Why were we not told?!?!?!?

Red Flag #3: This means that SIX members are departing the committee in 2014. That means that a majority of the committee (6 of 11) will be new members this year. In addition, two members were added in 2013 (Ms. Collier in May and Dr. Kaplan in October). And Dr. Nancy Lee currently provides no orientation whatsoever for new members. NONE.

Red Flag #4: Dr. Dimitrakoff was assumed by many advocates to be the heir apparent to replace Dr. Marshall as Chairman. Now it appears that the Chairman will be selected from the five remaining members: Dr. Casillas, Dr. Corbin, Dr. Fletcher, Dr. Kaplan, or Ms. Collier.

The CFSAC is being eviscerated. A majority of the committee will be new this year. Two of the five “veterans” will have served only a year. Of the five “veterans,” only one can be considered an ME/CFS expert, meaning that a significant portion of his/her time is spent on ME/CFS research or clinical care.

I shudder to think what this Committee will look like by the end of 2014 (assuming the charter is renewed in September, of course). Several steps must be taken to mitigate the risks to the ME/CFS community: the six new appointees must be ME/CFS experts and all of them should receive substantial orientation so they can hit the ground running.


Silver Platter of Frustration

March 12th, 2014 14 comments

Yesterday’s CFS Advisory Committee meeting was insane. Wait, maybe the meeting just drove me insane. Or was the whole thing just insanely inane? I don’t even know anymore. Wait a second, hang on.


Ok, let me start again.

Yesterday’s CFS Advisory Committee meeting served up a generous helping of frustration on a silver platter. While some of the mistakes from the last meeting were corrected, many mistakes were repeated and new ones were made. I’m going to be as succinct as possible in summarizing another episode of Tech, Wreck and Waste.

Webinar 101

Let me make this very straightforward and very simple: Do not run a webinar if you cannot make a webinar run. Here’s a checklist:

Can you provide clear audio? Some speakers were unintelligible. Dr. Sue Levine’s audio kept cutting out during her presentation. And for seven minutes (I timed it), the audio cut out completely. The closed captioning was not an adequate substitute, but did provide comic relief with such gems as translating “criteria” as “cry tears.”

Do you know how to use the slides? I really expected this to be nailed down after the fiasco that was the slide portion of the December meeting. But I was wrong. There were nine minutes (I timed it) at the beginning of Dr. Dane Cook’s presentation during which we listened to dead air followed by a discussion of whether members could advance the slides themselves, which buttons to push, which slides they were seeing, and so on. From this point on, the slides periodically caromed out of control, moving backwards and forwards to the point where I got dizzy and had to look away from the screen. Several times, the slides disappeared completely.

Have you secured your dog in another room? I love dogs. I own a big lug of a dog, and I know that you cannot always control what your dogs do or when they will decide to bark their fool heads off. Which is why, if you are speaking on a webinar, you should arrange for your dog to be in another room. It was hard enough to follow the sometimes chaotic discussion without distractions like background noise.

Have you anticipated technical difficulties and rehearsed ways to fix them if they arise? Slide problems. Sound problems. Conferencing people in and out problems. This went a little better than December, but still, it really isn’t rocket science to practice solutions in advance.

If you answered “No” to one or more of these questions, you are not ready to run a webinar.

The tech problems have real consequences for the public trying to follow the meeting. We don’t know who is speaking (or even who is present), the slides do not always advance with the discussion, and sound problems mean we can’t hear some discussion at all. It was very clear that CFSAC members are equally frustrated by these difficulties. In my opinion, the webinar format should be abandoned until these technical issues are solved.

Stupid Questions

I believe there is really only one kind of stupid question: the question you do not ask. And there were some doozies.

  1. Not a single question for FDA about the Draft Guidance to Industry document. If I could read it and come up with a list of questions, why didn’t CFSAC members?
  2. Not a single question for AHRQ about the systematic evidence review. The evidence review is not only the cornerstone of the P2P Meeting, it is arguably just as significant (and long-term in its implications) as the IOM study. I have a looong list of questions about it. But maybe that’s just me.
  3. Little discussion about Dr. Cook’s presentation from the research and clinician-scientist recruitment working group. It seems like a lot of work went into that, and there were many potential topics for discussion. But from my notes, it looks like 15 to 20 minutes of discussion occurred.
  4. Not a single question for CDC, despite an issue that demanded strong questioning. (see the next section)
  5. Not a single question about the CFSAC charter renewal process.
  6. Not a single question about the appointment of a new Chairman.
  7. Not a single question about the timeline for appointing new members.
  8. Not a single question about what HHS is doing to ensure the coordination of the multisite study, P2P process, and IOM study – or even why these are all being pursued simultaneously to begin with.
  9. Not a single questions about the status of the High Priority Recommendations, and whether any have been completed.
  10. Not a single question about the status of adding links to ME/CFS organizations on the Office of Women’s Health website.

I Call Shenanigans

keep-calm-and-call-shenanigansDr. Sue Levine and the medical education working group were justifiably critical of CDC’s CFS website. Dr. Levine even suggested that someone investigate the potential for legal action against CDC to force some movement on the changes CFSAC has repeatedly recommended. At a minimum, she advocated that CFSAC identify who is responsible for the website in order to identify and deal with the roadblocks.

Dr. Belay (who had not responded during any of the roll calls so I’m not sure when he joined the meeting) jumped in to say that CDC has extensively revised the website based on committee input. The TookKit has also been revised, although he admitted that CDC had not taken down the old version as recommended by CFSAC. Dr. Levine asked what was causing the delay in making changes, and Dr. Belay responded that “we’ve made the changes a few months to a year ago.”

This is not true, as any CFSAC member could have established very quickly.

Denise Lopez-Majano checked the CDC website, as each page identifies when the content was last reviewed. The homepage? May 2012. General information page? May 2012. CDC CFS Publications? April 2012. Continuing education? July 2012. Case definition? May 2012. Symptoms and Causes and Diagnosis and Management? May 2012. The ToolKit? September 2011.

So was Dr. Belay simply mistaken, and the 2012 updates reflect the revisions made with CFSAC’s input? Or are the changes still trapped in CDC internal review? I have no idea. Someone should have asked.


I asked my husband last night if it was reasonable for senior-level people to present rough draft recommendations for a full committee to wordsmith together. He said he would be fired on the spot if he did that in his field. But wordsmithing by committee is precisely what happened for roughly two hours of the CFSAC meeting.

wordsmith1It wasn’t clear from Dr. Levine’s presentation whether she drafted the recommendations on her own, or if the working group had collaborated on drafting them. Whatever the working group’s process, it was abundantly clear that the draft was not ready for prime time, thus leading to the two hours of refinement.

Lack of clarity was pervasive throughout the recommendation language. What disease are we trying to educate doctors about? How should we define integrative medicine? Do we mean physicians or medical professionals? And on and on and on. The committee spent two hours hammering out all this stuff that could have been done partially in advance. FACA requires that the recommendations be discussed and approved in public. It does not require that they be written by the full committee in real time during a public meeting. There is no reason why the working group could not have spent two hours working out the details and supporting evidence, and then present a more polished version to the full committee. Non-working group members would still have a chance to ask questions, offer changes, etc.

I’m not saying the refinement was poorly done. The final version approved by the committee was significantly improved by the group effort. It was essential to replace verbs like “suggest” and “support” with verbs like “recommend” and “fund.” It was also essential to identify what supporting documentation and evidence should be submitted to the Secretary with the recommendations. My point is that these things could and should have been done before presentation to the committee. Not only was it frustrating and inefficient, but the time spent on this process meant that there was NO time for discussion of future issues for working groups and recommendations. A very large item of business was left unfinished.

So what did the committee actually recommend? Basically, the committee recommended that HHS fund the development of curriculum at medical schools, fund teaching modules featuring complex cases, support integrative medicine programs featuring learning about ME/CFS, fund novel programs to bring expert care to under-served areas, gather requisite data for established organizations to incorporate ME/CFS in education, and support the CFSAC effort to amend the CDC website. All of these recommendations were explicitly worded to focus on ME/CFS as defined by the 2003 Canadian Consensus Criteria.

What was missing was a statement of the case. Yes, multiple supporting documents were identified, including the 2003 Canadian Consensus Criteria, the Primer, and the Expert Letter to the Secretary. But the Secretary is (or should be) already familiar with those documents. HHS has already declined to follow the Expert Letter or to remove the CDC Toolkit. Why should the Secretary listen now? In order to create a compelling argument for these recommendations, the working group should have prepared a one page statement of the case. That case could present the data on medical school education and the responses the working group got when they contacted the professional associations (which boiled down to “prove to us this is a public health problem”). They should be sending the Secretary a few paragraphs that convey not only the urgent need for better provider education, but also why the current efforts are inadequate. Instead, the committee is apparently deferring that to Dr. Marshall, who will write the cover letter accompanying the recommendations. Will everyone on the committee be satisfied with what he writes? I hope so, since they delegated the task to him and did not ask to see a draft version before it goes to the Secretary.

Widening Divide

The public comments raised an issue that is increasingly troubling to me. Dr. Jon Kaiser (founder of K-PAX Pharmaceuticals) closed his remarks with strong praise for all the federal agencies and their efforts on ME/CFS. Bob Miller cited four examples of how he sees the federal government “turning a corner” on ME/CFS, although he pointed out that results will be the ultimate measure of success. The rest of the public comments took HHS and CFSAC to task for lack of progress, or worse.

There has always been a divide in the ME/CFS advocacy community between advocates who thought the government was making progress (albeit slow and inconsistent) and those who thought the government was stalled or moving backwards (perhaps intentionally). But it seems to me that this divide has grown significantly wider in the last year. I’ll be writing more about this soon, so I’ll just put a pin in the topic to save it for later.

The Silver Platter

The disconnect between the accountability and progress that ME/CFS patients deserve and the decisionmaking put on display at CFSAC meetings remains large. These meetings are so frustrating, and increasingly so, that it is easy to see why some people believe HHS is doing this on purpose. Maybe they blame individuals, maybe they blame the Department, maybe they blame a highly placed policy maker, but many ME/CFS advocates believe that the sheer volume of problems can only be explained by intentionality.

WhitegloveSilverPlatterSizedHow else can we explain a repetition of technical difficulties from the December meeting? How else can we explain the CDC’s failure to be forthcoming about their own website? How else can we explain the conduct we see in these meetings, and the way CFSAC’s recommendations are handled by the Department? How else do we explain the lack of urgency?

I have no explanations to offer. But somebody could, and should. FDA has consistently demonstrated over the last two years that it is listening to patients and advocates. FDA has held open teleconferences and given advocates the freedom to ask questions and make their points. FDA held the public meeting last year, and followed through on its commitments to produce summary reports and draft guidance to industry within a year. Advocates do not agree with all of FDA’s decisions by any stretch of the imagination (e.g. Ampligen), but we recognize that FDA is listening and moving forward.

That is what progress looks like. And the contrast with CFSAC could not be more stark or more troubling.


More on P2P

January 13th, 2014 21 comments

Robert Miller posted a statement on Facebook last night revealing that he was one of the members of the P2P Working Group that met at NIH last week. I’ve posted his full comment below, with his permission.

Bob is very positive about the meeting and P2P process. I’ve spoken with several people who attended this meeting, and I have heard mixed reactions. Some are positive, some quite negative. The members of the Working Group are all, as Bob says, some of our best experts. Bob says that NIH listened to their input, and that the Working Group “drove the agenda” for the P2P Workshop. Again, I have not heard the same optimistic assessment from everyone who attended the meeting.

As I said before, there are two fundamental problems with the P2P process:

  1. The P2P panel cannot – by design – include anyone who has ever published on ME/CFS or taken any position on it. The Workshop could be exactly as Bob and the other Working Group members designed, but non-experts will do the evidence review and non-experts will comprise the entire Panel. That Panel will write the final recommendations, not the Working Group or meeting presenters.
  2. There is no transparency. I am very glad Bob came forward to acknowledge his participation in the Working Group. But the questions they finalized at the meeting have not been made public. The roster of the Working Group has not been made public (although word is leaking out). My understanding from two people who attended the meeting was that the discussions at the meeting were confidential. While Bob and the Working Group nominated potential panelists, the actual selection process will be done in secrecy. We will have no input, and no idea who those panelists are until the meeting is about to happen.

This is not acceptable to me.

We are constantly admonished not to question the motives of the people involved in these efforts. I do not question anyone’s motives, nor have I seen evidence of a conspiracy. I agree with Bob’s view that we should engage in these issues in a positive and professional way. My advocacy “career” is based on those values.

But a P2P Panel that will not include any ME/CFS experts? A Panel that will be selected behind closed doors? An evidence review conducted by non-experts? And the outcome of the process is a series of recommendations on diagnosis, treatment and research? No, no, no, and no.

The ME/CFS advocacy community would never have accepted an IOM committee that had no ME/CFS experts on it. We already know that the P2P panel will involve no ME/CFS experts. I do not accept this, and neither should you.

People have asked me what we can do about it. I am actively pursuing several options, and I will keep you posted.

Here is Bob’s statement from Facebook last night:

A brief update for everyone: last week I was invited as a patient representative to the NIH Working Group meeting for the NIH Pathways to Prevention Workshop on #MECFS (happening in the future). This is what was described as an Evidence-based Methodology Workshop at last Spring’s CFSAC meeting. I was asked at the last minute because the original patient advocate could not attend. The Working Group was charged with preparing questions for a thorough evidence-based literature review to identify gaps in ME/CFS scientific research, and we recommended expert speakers and independent panel members for the workshop itself. You can find details of the P2P program below. It is an independent scientific review, and the same process has been used before with another illness.

I want everyone to know my perspective. The Working Group was composed of some of our best experts, and I developed real respect for the person who will Chair the independent panel. Our experts and I had real input into the agenda and questions. The Working Group drove the agenda, and we will participate in the Workshop. I believe the prep work for the Workshop is being done with strong representation from our illness, laying the foundation for a good outcome.

I have been pretty ill in recent months, so I have been stingy with my energy. It has been difficult to post a lot about what is happening in our illness. With this NIH Workshop in mind, and the other governmental initiatives occurring in ME/CFS, I want to encourage patients to engage positively in federal work on our illness. We have had 25 years of inaction by the federal health agencies, and that hasn’t been good for us. All of us have asked for a serious commitment to ME/CFS by the federal government. That is what President Obama promised my wife. These initiatives – the FDA Drug Development Workshop, NIH Pathways to Prevention Workshop, Institute of Medicine Diagnostic Criteria Panel, the CDC’s 5 year Clinical Assessment Study – are all steps toward a stronger federal response. All of these initiatives are not an accident – they are the result of years of work by many of our patients and advocates, to change the federal approach for the better. Patients educated the FDA last spring in a way that has never happened before, and we have the same opportunity at the IOM meeting coming up. The IOM has strong representation on the ME/CFS Committee because patients engaged in it. We need to mix our expert clinicians & scientists with new experts in relevant fields of biomedical research to change our health. I welcome all of these initiatives and know that we will have to do hard work on the details. We won’t agree with everything in these processes or outcomes, but we need government support and action to improve diagnosis, treatment and understanding of ME/CFS. I continue to believe we need to Unite to make the most of new government attention and that 2014 will be a turning point in so many ways. Happy New Year to every one who suffers from ME/CFS. I promise we will move forward together in 2014!

Behind Closed Doors

January 6th, 2014 50 comments

SecretsThere’s an important meeting happening at NIH today and tomorrow, but you probably know nothing about it. The secrecy of this meeting is intentional, and the implications of decisions made at the meeting are as far-reaching as the Institute of Medicine study. In fact, what I’ve learned about the meeting may strike you as worse than the IOM study process.

TL;DR Version

  • The P2P Working Group roster has not been made public.
  • The P2P Working Group will finalize the study questions for the evidence review and workshop.
  • I can exclusively reveal who was contracted to conduct that evidence review.
  • I can exclusively reveal the draft study questions.
  • The P2P panel, which will conduct the Workshop and write its report, will be 100% non-ME/CFS experts.

What Meeting Is This?

January 6-7th is the first meeting of the Working Group for the Pathways to Prevention Workshop on ME/CFS. You may be more familiar with the old name for the meeting, the NIH Evidence-based Methodology Workshop. At the May 2013 CFS Advisory Committee meeting, Dr. Susan Maier clarified the purpose of the Workshop “is to identify methodological and scientific weaknesses in a scientific area and move the field forward through the unbiased and evidence-based assessment of a very complex clinical issue.” The Workshop itself will not create a research definition for ME/CFS, but Dr. Nancy Lee said that the output of the workshop could be used to inform such a definition. (CFSAC Minutes, May 23, 2013, pp. 6, 48-49)

The Pathways to Prevention Program (P2P) is operated through NIH’s Office of Disease Prevention. Each workshop process takes about a year from start to finish, and its foundation is a technical brief providing “an objective description of the state of the science, a summary of ongoing research, and information on research needs.” This brief is prepared by one of the Agency for Healthcare Research and Quality’s (AHRQ) Evidence-based Practice Centers (EPC).

At today’s meeting, the Working Group will finalize the study questions that frame the entire workshop process. Obviously, the questions are of critical importance since they form the basis for the evidence review and technical brief, as well as the public workshop itself. But before we get to the questions, don’t you want to know who is on the Working Group?

Who Is On This Working Group?

Guess what? We don’t know. At the May 2013 CFSAC meeting, Dr. Maier reported that 35-40 potential names were forwarded to the Office of Disease Prevention for possible service on the Working Group. She said that the list included ME/CFS experts, advocates and patients, including some CFSAC members. Despite vigorous objections expressed by Dr. Mary Ann Fletcher, Dr. Maier did not share the list, did not allow CFSAC to provide input, nor did she indicate a timeline for the release of that roster. (CFSAC Minutes, May 23, 2013, pp. 8, 49)

Unfortunately, Dr. Maier also did not provide the roster at the December 2013 CFSAC meeting and, to my dismay, no one on CFSAC asked her about it. Dr. Maier has also refused an individual request for the roster prior to the meeting, citing “standard procedure,” and there is no indication whether or when that information will be made public.

Why is this a big deal? Because the Working Group helps shape the entire workshop process. According to the P2P site, “the Working Group is involved from the beginning to the end of the workshop planning process; it finalizes the questions that frame the workshop, nominates panelists and speakers, and selects the date of the workshop.” Interestingly, the Working Group is made up of “content area experts from the federal government, academia, and clinical practice.” Dr. Maier said the nomination list included advocates and patients, but we have no way of knowing if any were actually appointed to the Working Group.

The Study Questions

Dr. Beth Collins Sharp described the evidence review process in detail at the May 2013 CFSAC meeting. One of the AHRQ EPCs is contracted to conduct a comprehensive evidence review based on study questions. Those study questions were drafted by unknown federal employees, and are finalized with the input of the Working Group, the EPC and federal participants. This is happening today and tomorrow.

As Dr. Collins-Sharp said in May, “You can’t get the right answer if you don’t ask the right questions.” (CFSAC Minutes, May 23, 2013, p. 12) However, Drs. Maier and Collins-Sharp have refused an individual request for the study questions being presented to the Working Group today, and have said only that the final questions will be posted by AHRQ and ODP but provided no timeline for this. Incidentally, they have also refused to disclose which EPC was contracted to perform this review.

However, I can answer both those questions today because I obtained a copy of the EPC task order through FOIA. The “Small Systematic Review for Diagnosis and Treatment of Myalgic Encephalophyelitis/Chronic Fatigue Syndrome (ME/CFS)” will be conducted by the Oregon Health & Science University for $358,211. I will discuss this contract in more detail in a future post. For now, I draw your attention to the draft questions as stated in the Task Order, and presumably being presented to the Working Group today:

I. How do ME and CFS differ? Do these illnesses lie along the same continuum of severity or are they entirely separate with common symptoms? What makes them different, what makes them the same? What is lacking in each case definition – do the non-overlapping elements of each case definition identify a subset of illnesses or do they encompass the entirety of the population?

II. What tools and measurements will help define the subsets of individuals in the entire spectrum on ME/CFS? Are some of these tools already available and can they reliably detect a subset of illnesses? Is it possible to identify which subset of individuals is not defined by the current tools and measurements? What is unique about the illness quality in those individuals not captured by the tools available?

III. What are the characteristics of the individuals who respond to specific treatments in terms of the spectrum of the disorder? Why do some individuals not respond? What aspect of the disorder is most relieved by specific treatments? For treatments that have been shown to be effective, what are (is) the underlying mechanism(s) of action of that intervention?

IV. What does clinical research on ME/CFS tell us about clinical diagnosis of ME/CFS? Are there hints in the current literature for a consistent defining characteristic? In the clinical realm, what differentiates borderline “cases” into confirmed versus probable? Do co-morbidities help define subsets of the clinical cases?

V. Have previous research findings shaped current clinical practice or are research and clinical practice completely separate with respect to the illness? How much influence does biomedical research help shape [sic] the future of ME/CFS research?

There are so many issues with this list. For starters:

  • Asking whether ME and CFS differ is critical (I), but this question fails to ask whether the mixed bag of “CFS” is even a valid clinical entity to begin with. It’s also important to note that the remainder of the questions (II-V) revert to lumping ME and CFS back together as one illness.
  • Question II asks what tools/measurements can be used to identify subsets along the whole spectrum, i.e. NOT whether such a “subset” actually represents a separate illness. It also asks if there is a subset not defined by current tools and measurements. Huh? How could a subset be identified if there are no tools/measurements to identify them?
  • Question III, the characteristics of patients who do or do not respond to treatment, rests in part on case definition. Will a systematic review dig into the raw data on studies such as the PACE trial or Ampligen trials to identify characteristics of responders and non-responders? Can applicable case definitions in those study cohorts even be assessed retrospectively (e.g. to examine a Fukuda cohort to see how many met the Canadian criteria)? Will the systematic review treat studies with different case definitions as equivalent (e.g. Oxford studies are as valid and relevant as Fukuda studies)?
  • Question IV strikes me as the question actually being posed in the IOM study. The IOM will be identifying the evidence for various diagnostic criteria, and then develop evidence-based clinical diagnostic criteria.  Including the same type of question here seems needlessly duplicative. And what if the two evidence reviews come up with different answers?
  • Finally, I can answer Question V myself: it’s both. There are a number of key clinician-researchers who maintain a clinical ME/CFS practice and conduct research. For those individuals, their research influences their clinical care and vice versa. But for the rest of the world, we know that clinical care is completely divorced from ME/CFS research. Based on the horror stories we hear from patients, based on the dramatic under-diagnosis of the disease and simultaneous use of CFS as a wastebasket diagnosis, I think it is abundantly clear that research and clinical practice is separated by a great wall for most patients.

The Working Group’s planning appears to be closed to the public, and we have no input onto the final questions. We wouldn’t even have this draft list if I had not managed to file a successful FOIA request. The anonymous Working Group will finalize the questions, and these will be posted publicly – although we have no timeline for that.

Non-Experts By Design

Supposedly, the Working Group is made up of ME/CFS experts. That’s the impression Dr. Maier gave at the May 2013 meeting, and by the P2P website. But the P2P Panel is a completely different story.

The P2P Panelists perform several functions: review the evidence report produced by the AHRQ review; attend a pre-Workshop webinar to discuss the evidence report and additional materials; attend the Workshop and ask questions of the presenters; prepare a draft panel report; and review and incorporate public comments to finalize the report.

Panelists can be nominated by members of the Working Group BUT there are significant restrictions on their expertise. Specifically, the panelists:

  • May be knowledgeable about the general topic under consideration, but must not have published on or have a publicly-stated opinion on the topic.
  • Must not have intellectual conflicts, such as participation in statements by professional societies or participation in advocacy groups on the topic.
  • Must not hold financial or career (research) interests in the workshop topic.

keep-calm-and-bang-your-head-against-the-wallLet’s be very clear about what this means. If someone has ever published on or made a public statement about the diagnosis and treatment of ME/CFS, he/she is disqualified. If someone has participated in statements from professional societies or participated in advocacy groups, he/she is disqualified. If someone has a financial or research interest in the diagnosis and treatment of ME/CFS, he/she is disqualified. Furthermore, there is no public comment period on the panel roster like there was for the IOM panel. In fact, it’s not even clear to me how far in advance we will know who has been appointed to the panel.

If the IOM process makes you mad, then this process should make you furious. There will be no ME/CFS experts on the panel that writes the Workshop report identifying methodological and scientific weakness in ME/CFS, suggesting research needs, and providing “an unbiased, evidence-based assessment of a complex public health issue.” The only involvement of experts will be through the Working Group and through the presentations made at the Workshop. I only see one upside to this arrangement: anyone who has been associated with the psychogenic model of ME/CFS will also be excluded.

This process may work very well for the “generally noncontroversial topics” that P2P is designed to address. For example, I can easily imagine the benefit of non-experts examining the state of research for a rare genetic disease. Only one other disease has been examined through P2P: polycystic ovary syndrome. The P2P workshop examined the current diagnostic criteria, causes and risk factors, and prevention and treatment strategies. There were only four panel members: an obstetrician-gynecologist, a cardiologist, the executive director of the American College of Nurse-Midwives, and the Executive Dean for Research at the Mayo Clinic. No patients or advocates spoke at this Workshop. It is not clear to me how well received the panel’s recommendations were in the PCOS patient community.

There are obvious problems with trying to apply this process in ME/CFS. First, there is no single body system to focus upon. While the PCOS Workshop could draw on endocrinologists, gynecologists and women’s health experts, what is the specialty pool for ME/CFS? Second, it is well known, and I believe generally accepted, that doctors and researchers without ME/CFS expertise will still have preconceptions about the disease. We need look no further than FDA for an example. It wasn’t until after the four-hour active listening session in April 2013 that FDA representatives, by their own admission, began to understand the seriousness of the disease, and this was a group of people who were familiar with ME/CFS to some extent. If the P2P panel is comprised of people with little ME/CFS knowledge and possibly negative preconceptions, and the Workshop does not include significant participation from ME/CFS patients and advocates, it seems unlikely that the best results will be achieved. Based on our decades of experience with misinformed scientists, clinicians, and policy makers, it is very hard to trust in such a process.

Bottom Line

Almost the entire process of this Workshop is being conducted behind closed doors. The Working Group roster has not been released. The Working Group meeting is not open to the public. The draft questions were obtained only through a FOIA. There is no information about when the final questions will be posted. We have no idea who will be on the Panel, or even who will make that decision. And the only way ME/CFS experts are likely to participate is through the Working Group (IF there are any on the Working Group) and through presentations at the meeting. The extent to which members of the public will be able to participate is completely unclear.

So if you are worried about the lack of ME/CFS experts on the IOM panel, or if you think that the public will not have a sufficient opportunity to participate in the IOM process, pay attention! The NIH P2P process looks even worse. We cannot lose sight of the forest for the trees, and IOM is not the only moving piece on this chessboard.

What can we do? I believe that advocates must demand more information about the P2P Workshop, and must demand meaningful opportunities to participate. The planning and execution of the Workshop should be transparent if it is to have any legitimacy in the advocacy community. I urge you to participate in both the IOM and P2P processes at every opportunity – ask questions, provide input, and present a united front based on the truths we know about ME/CFS. We cannot wait until the end of the P2P process to make our voices heard, especially since this process will provide input into the IOM study.


Need to Reality

August 5th, 2013 7 comments

One of the key moments of the April FDA meeting on drug development for ME/CFS was when Bernard Munos said that ME/CFS patients will have to collect and pool their data to attract the interest of big pharma. Many advocates were dismayed by that comment. How can we collect our data? We have no resources. We’re too sick!

Enter PCORI. The Patient Centered Outcomes Research Institute was created by the Affordable Care Act, and is a non-profit organization funded by the federal government. Their budget is huge: $320 million for FY 2013 alone. And they fund research, lots of it. In May 2013, PCORI issued a funding announcement for patient-powered research networks and it could not have come at a better time. Up to $1 million per project is available to help fund data infrastructure to collect and pool electronic data from patients in order to facilitate clinical outcomes research, which is exactly what Munos told us was needed.

Two different ME/CFS projects were proposed, one led by the CFIDS Association and one led by the Open Medicine Institute. In full disclosure, I was one of many advocates consulted by the CFIDS Association in preparing their Letter of Intent, but I have no formal affiliation with the project. Both projects were supported by multiple patient organizations. Neither the CFIDS Association nor OMI were willing to tell me on the record if they were invited to submit a full proposal.

Both organizations already have the beginnings of patient centered research networks. The CFIDS Association has a nascent research network through the SolveCFS BioBank. For the PCORI proposal, the CFIDS Association went big and partnered with Patients Like Me, one of the largest patient-powered research networks in the country. Patients Like Me has almost 10,000 patients in the ME/CFS community on its site, and has the big data expertise to create systems to capture both clinical data/electronic health records and patient reported outcomes. OMI has been building its own online patient data system. They recently received a grant to build a “new personalized medicine infrastructure.

PCORI has set aggressive goals for the projects it funds under this opportunity. During the 18 month period of grant, funded projects must achieve the following: Membership must reach 0.5% of the US population with the condition; patient-reported data must be collected from at least 80% of the participating cohort, patients must be fully involved in network governance, and data must be standardized and suitable for sharing with other infrastructure members.

These patient-powered research networks are not just big databases sitting in server rooms. Patients themselves must be in control of the data and governance of the network. This would be a first in the ME/CFS community: none of the existing biobanks and databases are governed by patients. Traditionally, the non-profits and researchers have determined the features of these databases and how they will be used. But PCORI is requiring direct governance by the network participants themselves. This will be a unique challenge for either proposal. Getting back to the advocates’ reaction to Munos, we’re too sick! So few of us have the capacity to serve on steering committees and boards of directors, and those that have capacity are already involved (often in multiple contexts). A proposal from the Association or OMI will have to think beyond traditional forms of governance in order to collect the input and opinions of the network membership.

Another challenge will be privacy and confidentiality. Patients Like Me pools anonymized data and sells it to partners, and OMI is a for-profit health practice. Participants in either network should pay attention to the consent agreements and understand exactly what will be done with their data. PCORI networks will collect more than just demographic data. Other collected data will include patient-reported outcomes instruments and electronic health records. Managing and protecting the privacy of participants will be one of the challenges for network governance.

So what’s next? Full proposals for PCORI funding are due September 27, 2013 and funding would begin in January 2014. Let’s hope that at least one of the ME/CFS proposals is successful. One million dollars would go a long way towards building what Munos said we needed: a patient-powered network of data that can be shared with researchers and form the basis of treatment trials and biomarker identification. If there is any disease cohort that needs this kind of jumpstart funding, it is the ME/CFS community.

Update August 13, 2013: The CFIDS Association announced on its Facebook page that it has been invited to submit a full application. Dr. Suzanne Vernon said in an email to me, “The CFIDS Association partnered with 10 organizations and were invited to submit a full application to compete for $12 million that will fund 18 patient-powered research networks.  Our full application is due the end of September and organizations are notified of awards in December.  I am hopeful we will have a very competitive application!”


No Facts for YOU!

June 6th, 2013 14 comments

The NIH funding argument is a broken record: Advocates and researchers say, “We want more funding!” NIH replies, “We need more applications!” And advocates and researchers reply, “Your review panel is made up of dentists!”

Back and forth. Back and forth. Dr. Nancy Klimas said at the April FDA meeting that only 1 in 8 of her applications to NIH for ME/CFS research are approved – a 12.5% success rate. In contrast, Dr. Susan Maier (NIH) reported to the CFS Advisory Committee that the 2012 acceptance rate was 18%. To my knowledge, she has not presented data on the historical acceptance rates, but she said at the May 2013 CFSAC meeting that she does have those numbers. Dr. Maier literally begged the CFSAC audience to increase the numbers of applications. Dr. Mary Ann Fletcher repeated the complaint that grants are reviewed by the wrong people. Back and forth. Back and forth.

What truly bugs the crap out of me about this argument is that these are factual questions with factual answers. The historical acceptance rate for ME/CFS grants is something that can be measured, as can the number of applications. Why aren’t we having the discussion based on data? Multiple researchers have said on multiple occasions that there is no point in submitting an application for ME/CFS research to NIH because the CFS Special Emphasis Panel that reviews these applications is not staffed by ME/CFS experts. But again, this is a factual question! Who serves on the CFS Special Emphasis Panels? How many of them are ME/CFS experts? Instead of repeating perceptions, let’s examine the FACTS. I have attempted to do just that, but have been thwarted by NIH policy. It’s time to tell you that story.

Last year, I exchanged multiple emails with Dr. Susan Maier and Don Luckett at NIH in an attempt to get the CFS SEP rosters. Some of those rosters were posted on the Center for Scientific Review website, but most were not. On October 19, 2012, Mr. Luckett emailed me:

We no longer post this roster online due to threats some previous panel reviewers have received. Anyone who wishes to view the roster is advised to submit a Freedom of Information Act (FOIA) request to the NIH FOIA office.

Based on that advice, I filed a FOIA request on November 1, 2012 requesting 1) the complete membership rosters for each meeting or other activity of the CFS SEP for the last ten years and 2) documentation in NIH’s possession relating to threats or alleged threats made against one or more member of the CFS SEP in the last 10 years. My intent was to evaluate the rosters to see how many experts participate, and to understand the nature of these alleged threats. Many readers are no doubt familiar with allegations of threats against certain researchers in the UK, so I think it is important to establish the facts behind the allegations – especially since these allegations are the stated reason for requiring a FOIA request for the SEP rosters.

On February 11, 2013, the NIH responded to my request. They found 94 pages of records responsive to the two requests, but they only sent me four redacted pages. The remaining 90 pages were withheld in their entirety under the FOIA exception for “clearly unwarranted invasion of personal privacy.” Needless to say, I appealed this decision on March 7, 2013. The basis of my appeal is:

The only way for the public to know whether the CFS SEP is staffed with scientists with the appropriate expertise is for those rosters to be public, just as they are for the panels in other areas of research. It is no invasion of privacy to disclose the names of the individuals appointed to this advisory committee. I am not requesting anything that is not available to the public in the case of hundreds of other panels.

Furthermore, there is a significant public interest in documents related to the alleged threats made against previous panel members. Specifically, I was told that these alleged threats were the reason that the panel rosters were no longer available. NIH cannot refuse to release the rosters and then also refuse to release documents that may or may not support that policy decision. It is entirely possible that these alleged threats are merely rumor and hearsay, but there is no way for the public to know if no information is released. Given that the alleged threats are being used as the justification for withholding information that would otherwise be available, and that up until a few months ago was available, the public benefit of accountability and openness clearly outweighs the small potential harm of disclosing these documents.

I am not requesting anything unusual. Review committee rosters at NIH are public. You can go on the CSR website and see the names and office addresses for hundreds of advisory committee members across many disease areas. I’m not requesting special treatment – I am asking for information that is available to other interested members of the public. Instead, not only am I prevented from reviewing the rosters, I am prevented from seeing the evidence that is being used as the basis for denying me the rosters.

As a result, we cannot have a fact-based conversation about the CFS SEP. Is it populated with dentists and non-experts? Or has the composition of the SEP improved over the years? Knowing these facts are critical to a productive dialogue about whether it’s worth the effort for ME/CFS researchers to apply for funding. And because we are denied access to these facts, we are stuck in the back and forth argument of whether more applications will lead to more money. In trying to protect the privacy of reviewers, NIH is perpetuating the perception that ME/CFS grants are reviewed by unqualified scientists.

I am still awaiting the NIH’s response to my appeal.

Updated June 7, 2013: Several people have pointed out that the May 2013 SEP roster is available on the CSR website. This makes the denial of my FOIA request even more ludicrous, and I will be updating my appeal accordingly.


June 3rd, 2013 23 comments

The good news, I guess, is that we survived another CFS Advisory Committee meeting. The bad news is that much of what happened made no sense to me. Some excellent summaries of the meeting are available, including this very detailed recap from Phoenix Rising. I would like to tackle a few of the topics that had me shaking my head, or asking myself if my experience of reality is so at odds with the Committee’s. This post is long, and I apologize for that. Here’s my report card on these head-scratcher issues:

Meeting Mechanics

I know how difficult it is to prepare for and moderate two full days of contentious meetings, and I imagine it is more difficult to do so when the meeting will be held in public. However, I was struck by how poorly some of the administrative aspects of this meeting were conducted. For example, the procedure for meeting and comment registration is unnecessarily complicated for a patient population that struggles with multiple forms of cognitive dysfunction. I hope this can be simplified.

On the first day of the meeting, Dr. Gailen Marshall said that members would be limited to three minutes for comments during discussion. He did not enforce this limit evenly, including on himself. More than one observer noted how long his own comments were, and how he sometimes monopolized discussion. I happen to think that a Chairman needs leeway to cover certain topics, but sometimes this seemed excessive. Participation by other Committee members is very uneven: Eileen Holderman, Dr. Mary Ann Fletcher and Steve Krafchick speak most frequently; Dr. Jordan Dimitrikoff and Dr. Susan Levine fall somewhere in the middle; Dr. Adrian Casillas, Dr. Lisa Corbin, Dr. Dane Cook, and Rebecca Collier rarely if ever spoke. It did seem like the non-voting liaisons were integrated into the discussion, and had opportunities to ask questions and offer feedback.

I was very frustrated by the evident lack of preparation for even simple agenda items. For example, when approving the list of ME/CFS organizations for linking on the Office of Women’s Health website, the Committee did not have a list in front of them and they approved the criteria on Day 2, after approving the list on Day 1. One of the criteria was that organizations would consent to being linked, but on Day 1 Dr. Lee said that was not checked, so the criteria list on Day 2 had to be amended. It was a mess, and hard to follow. A similar lack of preparation was evident in the discussion of the High Priority List, as we’ll see. There is simply no good reason for the lack of preparation. Why didn’t a staff member type up the list of criteria and list of organizations, provide it to the Committee and post as a slide, so everyone knew what they were voting on? The disorganization and confusion wastes time, at the expense of other issues.

One mechanic that worked fairly well, in my opinion, was the audience Question & Answer. The audience questions led to some very significant discussion, especially regarding CDC. Some people feel that Dr. Marshall should not curate these questions, and I thought that the “answer” discussion tended towards domination by the Committee instead of actual answers to the questions. However, I think the value of this kind of interaction was very clear and I hope this will continue.

High Priority Recommendations

From a procedural perspective, we scored a small victory here. Dr. Marshall acknowledged that the list was not handled correctly last year. I pressed the Committee to devote an appropriate amount of time to discussion of the list, and I was shocked when they agreed. I don’t know what happened at lunch, or who said what, but apparently there was enough concern expressed that Dr. Marshall said they would move the discussion to Day 2. They discussed the list and how to use it going forward, and voted on it in public as required by FACA. Procedurally, they handled the issue correctly.

But the substance of their decision is perplexing, at best. No list was posted, and it did not seem like the committee members had a single piece of paper in front of them with the full list. Items were added – but without reading some of the additions into the record – and a process for removing items was discussed but not used. Dr. Marshall downplayed the fact that several recommendations had been altered from the original form, saying that “the spirit is there.” Dr. Lee said that the old recommendations chart would now be a historical document, and the High Priority list would be the working list. Dr. Marshall said that of the eight recommendations the Committee made last year, three were complete and the other five would be added to the list. But he did not specify which recommendations he considered complete, and no one asked him to be specific. I can make a pretty good guess, but we won’t know for certain until the final list is posted.

And amid all this back and forth, Dr. Marshall said that the list was never intended to prioritize one recommendation over another within the list. Everything on the list is of equal importance. No one questioned or objected to this. This may not seem like a big deal, but it is. By acquiescing to this equal priority description, the Committee created a situation where holding a disability workshop is of the same priority and importance as holding a case definition workshop. I don’t think most members would agree with that statement, but now they’re stuck with it.

So what did they select as the high priority recommendations? The Committee combined the original seven recommendations on the January 2012 list, five of the eight recommendations made in 2012, and two recommendations added by Eileen Holderman. These items are listed in chronological order. New recommendations will be added to the list automatically, and the Committee will have to vote to remove an item once they are satisfied with the response from the Assistant Secretary. I’ve drafted my best guess at the specific recommendations they approved (given the lack of precision on the 2012 recommendations), and I’ll post the official version when it becomes available. Is this list ever going to matter? Technically, I think it matters a great deal but I’m not sure the Committee shares that view.

Invisible Information

One of the things I said we should watch for at the meeting was any mention of the Ad Hoc Workgroup. Guess what? They never mentioned it once. This is one of the things that has me questioning if my reality is different from the Committee’s. Dr. Lee made such a point of talking about the Workgroup in 2012, and their report was published in March. But at this meeting? Total silence. It’s as if the Workgroup doesn’t exist. And not a single Committee member brought it up or asked a question. Why? What is the status of this group? What did the Committee think about the report? Did they even READ the report? Am I the only person who thinks this is strange?

The other thing that received almost no attention is the response from the Assistant Secretary to the Committee’s October 2012 recommendations. This was posted to the CSFAC website shortly before the meeting (pdf link). Again, I don’t understand why the Committee doesn’t simply review these at the outset. Instead, the only discussion was when Steve Krafchick objected that the response to the recommendation to hold a case definition workshop was not actually responsive (see the discussion on case definition below for more details).

The risk of ignoring or glossing over the official responses to CFSAC recommendations is that we miss opportunities to understand the basis for those responses. For example, buried on page 3 of the response is this statement: “To date, CDC has not been able to confirm the occurrence of outbreaks of CFS.” This leaves me wondering how in the world CDC characterizes the outbreaks at Incline Village and Lyndonville. But because the Committee does not discuss the responses, these questions don’t get raised. If I write to CDC or submit a question for the next PCOCA call, I will probably be ignored. But a CFSAC member could ask these questions and get answers on the record. Instead, this information – and the opportunity to learn even more – is effectively invisible, and it has no apparent effect on Committee members and discussion.

Oh CDC, You So Crazy

The CDC’s report of activities was ho-hum, dry, and devoid of much of interest. It was not until the Q&A sessions that we actually learned anything important.

The very first question was whether CDC would use the two-day cardiopulmonary exercise testing in phase 2 of its multisite study. Dr. Unger said that the clinicians in the multi-site study felt a two-day exercise test was “not advisable.” She elaborated that patients travel some distance to get to the physicians involved in the study and that a two-day test was not feasible. It was not clear to me whether the concern was the time required or the physical impact on the patients. I was very surprised that the clinicians (Natelson, Klimas, Peterson, Kogelnik, Bateman, Lapp, Podell) were the ones who advised against using the test because most (if not all of them) have used two day testing for some of their patients. Steve Krafchick pressed Dr. Unger, stating the importance of two day protocols for exercise and neuropsych testing in order to objectively capture the effects of post-exertional malaise. Dr. Unger said they would rely on questionnaires for functional outcomes and the clinicians’ observations of clinical course. Krafchick said it was a mistake to eliminate the testing, and asked if Dr. Unger had talked to Dr. Chris Snell. Dr. Unger said, “No.” My jaw hit the floor. How could it be that Unger has never talked to Snell about CPET? They’ve been at meetings together, including the recent FDA meeting where Snell gave a presentation on two-day CPET. I still can’t wrap my brain around this. Two day CPET provides objective evidence of metabolic dysfunction, post-exertional malaise and estimate of disability. CDC, how could you refuse to use this test?!

Another great question for CDC was whether the website would include a highlighted warning that exercise can be dangerous for ME/CFS patients. Dr. Belay answered that the website states exercise can exacerbate the illness, and Dr. Marshall asked about the equivalent of a black box warning. Dr. Belay said they could consider it. Dr. Fletcher followed up with a reminder that the Toolkit recommends exercise as a therapy, and Dr. Belay said CDC has revised the Toolkit and it is going through clearance. My jaw hit the floor again. Why didn’t Dr. Belay think to mention that in his routine report? It’s obviously of interest to the Committee since they recommended last year that the Toolkit be removed from the website. I guess Belay was planning to wait until the revision was complete, because when Steve Krafchick asked if Committee members could see it for review or comments Dr. Belay responded, “Why?” and “we don’t do that.” Seriously? Dr. Marshall pointed out comments could be useful, and Dr. Lee said they could send informational copies to interested members for feedback.

The Committee discussed a review of the CDC website, including the photos which portray people yawning at work, going for slow walks, etc. Several committee members (and many patients) feel the photos are misleading because they do not portray the seriousness of the illness. Dr. Unger responded that they want to portray a “positive” side. Seriously? To be frank, it is conversations like this one that make patients wonder what planet CDC lives on that they think there is a positive or lighthearted side to ME/CFS. The discussion moved into case definition because the CDC website and medical education material lists multiple criteria, including the maligned Oxford definition, even though CDC says it endorses and uses Fukuda. There was more discussion of the 2-day CPET, whether the Canadian Consensus Criteria is difficult to use, and whether there was enough information to endorse the Canadian Criteria immediately as many advocates insist. Unfortunately, and typically, there was no resolution on any of these issues and case definition raised its ugly head again and again.

A Rose By Any Other Name . . . .

So we come to case definition. In October 2012, the CFSAC recommended that the Secretary:

promptly convene (by 12/31/12 or as soon as possible thereafter) at least one stakeholders’ (Myalgic Encephalomyelitis (ME)/Chronic Fatigue Syndrome (CFS)experts, patients, advocates) workshop  in consultation with CFSAC members to reach a consensus for a case definition useful for research, diagnosis and treatment of ME/CFS beginning with the 2003 Canadian Consensus  Definition for discussion purposes.

And the Assistant Secretary responded in writing on May 1, 2013:

The National Institutes of Health (NIH) is convening an Evidence-based Methodology Workshop process  . . . to address the issue of case definitions appropriate for ME/CFS research. However, it will not cover in detail a clinical case definition. The Office of the Assistant Secretary for Health, Department of Health and Human Services, is actively pursuing options for a separate effort that would work in coordination with the NIH process, but result in a case definition useful for clinicians who see patients with symptoms that may be ME/CFS. . . . .

The EbMW consists of a thorough, unbiased evidence review of the literature related to clinical research outcomes compared across case definitions and culminating in a workshop composed of experts and patients. The workshop participants and panel members will use the evidence review to evaluate the strength of evidence for case definitions with the goal of identifying the most consistent outcomes. . . . The first organizational meeting for the EbMW on ME/CFS was held on February 19,2013. A timeline for the process is being developed.

The wording of the response is very important: the EbMW will address the issue of case definitions appropriate for ME/CFS research. That does not say they will identify the correct or new research definition – just that the issue of appropriate research definitions will be addressed.

These two paragraphs from Assistant Secretary Koh’s response translate as follows: 1) No, we will not have a stakeholders’ workshop as you recommended in October 2012. 2) We will have an EbMW to address definition issues related to research. 3) We are “actively pursuing options for a separate effort” on a clinical case definition.

Understandably, several CFSAC members were upset that the answer was No-but-we’ll-do-something-else. This is what led to the fireworks at the end of Day 2. Several members reacted strongly to Dr. Susan Maier’s report that the unidentified people who attended the meeting on February 9th submitted a list of 35 to 40 potential candidates for the EbMW’s organizational committee to the Office of Disease Prevention for vetting and selection. Dr. Maier could not identify that list of candidates, although she did say that there were CFSAC members and advocates on the list. We have no timeline for publication of this list, either. Dr. Fletcher was particularly vocal about the secrecy and long timeline

I have to say that I am not surprised that the answer was “no, but . . . ” and I’m not knocking the EbMW. Dr. Beth Collins-Sharp from the Agency for Healthcare Research and Quality gave a detailed explanation of the methodology used for evidence reviews, and it is quite robust (and includes a patient viewpoint). I suspect that this is the same kind of evidence review that was requested by the CFSAC years ago in order to have a State of the Science Workshop, and which was never completed. The State of the Knowledge meeting in April 2011 was basically a State of the Science-Light kind of meeting. AHRQ’s last review on CFS was completed in 2002, so it is certainly time for an update.

The problem here is that we don’t have enough information to judge the EbMW process, since we don’t know who is on the organizing committee. We can’t judge the case definition process, since we have absolutely NO information about it whatsoever. And this leads to the real problem: THIS IS TOO SLOW.

As Dr. Wanda Jones reminded us in her welcoming remarks on Day 1, government moves slowly. It does indeed, and this creates extraordinary frustration for every patient and advocate involved. It seems unlikely (at this point, anyway) that the Canadian Consensus Criteria will be adopted as an interim measure, and none of the other case definition processes will bear fruit within the next six months. The government apparently expects us to wait patiently and calmly as this process unfolds at a bureaucratic pace. We don’t have a choice about the waiting part, but I don’t think the expectation that we will be patient and calm is realistic at all. This issue is too huge, too important, too divisive, and too slow. People are angry and will continue to be so, unless the government can demonstrate urgency.

If anyone from HHS is reading this post, may I suggest that you improve the way you communicate around this issue as a first step? The FDA communicated openly with us, and also produced a great meeting. Look to FDA for ways to productively and positively engage the patient advocate community. If you don’t, we are likely to see fireworks of one kind or another at every CFSAC meeting going forward.