Clarity on Comment

Posted on March 28, 2013 by Jennie Spotila

As I said when the FDA formally announced the commenting process for the Drug Development Workshop, the process will be very different from what we’re used to from CFS Advisory Committee meetings. At the CFSAC, we sign up for a comment slot, submit our complete comments in advance, and then read those comments at the appointed time. There is no discussion, and time limits are strictly enforced.

The Drug Development Workshop will follow a very different format. Several advocates, including myself, have sought clarity from FDA on how it will work. We’ve received more details, and I’m trying to get the word out so people can choose how to participate.

You Must Register

You must register to attend the Workshop, regardless of whether you want to comment. The deadline to register is April 8th.

To Be On A Panel

On Day 1, discussion will start with two panels: “Disease Symptoms and Daily Impacts That Matter Most to Patients,” and “Patient Perspective on Treating CFS and ME.” If you would like to be on one or both of the panels, you must do the following:

  1. Indicate your interest in serving on a panel in your meeting registration.
  2. Send a brief summary of your responses to the panel topic(s) you are interested in to ME-CFS-Meeting@fda.hhs.gov by April 8th. FDA has told us: “We do not need lengthy responses or verbatim testimony. We are just looking for enough detail to make sure that the patients identified to kick off the discussion can represent a range of experiences with CFS and ME.”
  3. If you are selected for the panel, you will be notified in advance and will receive more details about how the format will work.

To Participate in Discussion

After each panel on Day 1, the floor will be open for discussion. This is completely different from the CFSAC format. Remember, you need to register by April 8th to be at the Workshop. Here is how the FDA describes the way discussion will work:

We will then open the discussion to include other patients in the audience who are interested in adding to the discussion. There is no need to register for participation in these audience discussions, and we are not asking for prepared testimonies to be presented as part of the audience discussion. We plan for our facilitator to ask prompting questions and call on people who raise their hand. We will try to accommodate everyone who wants to contribute to the discussion, and we will need to work within the meeting time constraints.

Got it? So to make comments after each panel, you simply need to raise your hand and wait to be recognized by the facilitator. This is NOT the time to comment at length or talk about a different topic. This is a group discussion focused on the questions FDA has posed in the agenda (pdf link).

To Comment on Another Topic

Approximately 30 minutes has been set aside at the end of Day 1 for open public comment. This will follow the format we are more familiar with. If you want to comment during this time, you must do the following:

  1. Indicate your interest in offering comments in your meeting registration.
  2. Send a brief summary of your comments to ME-CFS-Meeting@fda.hhs.gov by April 8th.
  3. FDA says: “To date, we have had a large set of people register to present in the open public comment period, and it will be a challenge to accommodate everyone within our time constraints. We encourage people to  use the open public comment period to address  topics that were not addressed by Topics 1 and 2 earlier in the day.“

You Can Always Comment in Writing

You can provide written comments to FDA either before or after the meeting (until August 2). To submit comments, go to the docket and fill out the form.

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Index of FDA Meeting Resources

Posted on March 25, 2013 by Jennie Spotila

One month to go until the FDA’s Drug Development Workshop for ME and CFS! There is a boatload of information that might be helpful to you in preparing for the meeting, even if you will only be watching online. I’ll add to this list as more resources become available.

 

Meeting Materials

FDA’s meeting materials page

Draft agenda (pdf link)

Why this meeting is important

FAQs about the meeting (pdf link)

 

How to Participate

If you want to speak at the meeting, comments are due April 8th

Register for the meeting and submit comments

Submit written comments to the docket (open until August 2, 2013) using the docket number FDA–2012–N–0962

The questions FDA would like patients to answer

Suggestions on what to focus on in your comments

CFIDS Association survey

Dr. Lily Chu and Dr. Leonard Jason’s survey

Register to watch the meeting by webinar

 

How FDA Evaluates Drugs

FDA’s Drug Approval Process

Drug Approval Process Infographic (pdf link)

CFIDS Association webinar: Overview of the Drug Development Landscape (March 28, 2013)

Patient Focused Drug Development Program

Designating ME/CFS as a serious or life threatening condition

Speeding up the process for drug approval

FDA’s webinar on advocacy and drug approval (November 2012)

FDA’s teleconference with ME/CFS patients (September 2012)

 

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Two Surveys, Two Opportunities

Posted on March 22, 2013 by Jennie Spotila

In addition to all the ways you can participate in and comment on the upcoming FDA Drug Development for ME/CFS Workshop, you can also participate in two surveys that will collect data for presentation at the Workshop.

The CFIDS Association is conducting a survey based on the questions FDA posed in the Federal Register notice for the Workshop. In describing the survey, the Association says:

The survey, designed for people living with ME/CFS and those closest to them, poses open-ended questions to allow respondents to describe the symptoms, daily impacts that matter most, as well as share personal perspectives on treatment. Questions are worded in the same way the FDA has asked them; we’ve supplemented with some some additional questions on the same topics. You can answer with phrases or lists; you do not have to write complete sentences.

All survey responses are anonymous and confidential. We intend to present an analysis of the collective responses at the FDA meeting and at the May 22-23 meeting of the federal CFS Advisory Committee to better inform the federal health agencies about these issues.

The survey will take an estimated 30-60 minutes to complete, and your responses are completely anonymous. Follow this link to begin the survey.

Dr. Lily Chu (who is serving with me on a panel at the Workshop) and Dr. Leonard Jason are also conducting a survey. Lily says:

The main purpose of this survey is to help answer questions the FDA has about ME and CFS symptoms, their impact on people’s daily lives, which treatments patients are using or have tried, and which treatments patients have felt to be effective. Your answers will help teach drug companies about the symptoms/ severity of these illnesses while considering new medications for treatment. I will present the results of this survey at the April FDA meeting.

Instead of asking you to type in answers to each of FDA’s questions, our survey allows you to choose specific answers to questions while allowing space for comments. This may save you some time/ energy typing and also remind you to think about certain symptoms, their impact on your life, and certain treatments. It also allows us to compare your answers to others with ME or CFS.

If you want your answers to be part of the results shared at the FDA meeting, please complete the survey by APRIL 17. The survey will remain open until May 10 though and all responses submitted will be included in our final report to FDA in August.

This survey will take an estimated 30-40 minutes to fill out, and your responses are completely anonymous. Follow this link to begin the survey.

You can participate in both surveys. In fact, I strongly encourage you to participate in both surveys. Each survey needs enough responses to make the data analysis meaningful. You can also participate in the surveys and still submit comments to the FDA. Again, I strongly encourage you to do so. As I said earlier this week, this meeting is a showcase and significant opportunity for the ME/CFS community to teach the FDA and drug developers about our disease. I hope you will invest time and energy over the next few weeks to participate in the surveys and speak directly to the FDA.

 

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Evidence Based at NIH

Posted on March 21, 2013 by Jennie Spotila

Last year, NIH said it was undertaking a process to identify a research case definition for ME/CFS, but individuals like me were unable to get any additional information about what NIH intended. Now additional information has been made public in a February 27, 2013 letter from Assistant Secretary for Health Dr. Howard Koh to Kim McCleary of the CFIDS Association (pdf link). I’ve done some research, and I believe we need to monitor this case definition process closely.

Case definition has been the 600-pound gorilla in the room every time ME/CFS is discussed. There are five definitions currently in use (Oxford, Fukuda, Carruthers 2003, Carruthers 2011, and Jason Pediatric) and there is no consensus among researchers and policymakers on which one is the best. (This is such a complex issue, it deserves its own post.) NIH, as the largest funding source for biomedical research, can wield great influence over the definitions used by researchers. While it was a positive step to say NIH would undertake a research definition effort, there was no transparency or information available to us until now.

In his letter to Ms. McCleary, Dr. Koh said:

the [Trans-NIH] Working Group submitted a competitive application for an Evidence-based Methodology Workshop (EbMW) on ME/CFS coordinated by the NIH Office of Disease Prevention. 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 goals of identifying the most consistent outcomes. The Working group successfully competed with proposals from other Working Groups across NIH (e.g. The NIH Pain Consortium) for funding provided by the NIH Office of Disease Prevention. The first organizational meeting for the EbMW on ME/CFS was held on February 19, 2013. (emphasis added)

It turns out that the NIH Office of Disease Prevention has a Consensus Development Program that produces, “evidence-based consensus statements addressing controversial medical issues important to researchers, healthcare providers, policymakers, patients, and the general public.” The Office of Disease Prevention provides infrastructure, funding, and coordination for the process. The process entails the following steps*:

  1. A Steering Committee of ME/CFS scientific/research experts and federal partners coordinates the EbMW with logistical support from the Office of Disease Prevention.
  2. A conference panel is selected. The panelists “give balanced, objective, and informed attention to the topic.” They cannot be HHS employees or have a financial or career interest in the conference topic. They may be familiar with the conference topic “but must not have published on or have a publicly stated opinion on the topic.”
  3. The Agency for Healthcare Research and Quality conducts a “systematic evidence review” and produces a report for the panel members prior to the conference.
  4. A two and a half day conference is held, consisting of presentations by subject matter experts, followed by discussion among the panelists, speakers and public in attendance.
  5. Dr. Susan Maier told me, “ME/CFS science/research experts and patient advocate groups with scientific/research expertise will be invited to participate in the workshop; the workshop is open to the public and will be videocast and archived.”
  6. The panel drafts a statement during the meeting and presents it for commentary on the third day. The final report is issued six weeks later. It is published online and in a major peer-reviewed journal.

The Office of Disease Prevention has recently followed this process for meetings on Polycystic Ovary Syndrome (conference held December 2012) and Diagnosing Gestational Diabetes Mellitus (conference held March 2013).  In scanning the materials for these two conferences, I was struck by the absence of patients or advocacy representatives on both the Steering Committees and Panels. I point this out not to raise a ruckus about it. Rather, I think we should educate ourselves about how the process has worked in other diseases, and prepare to operate within the constraints of the existing process. If EbMW never use patients as panelists, then we should accept that limitation of the method. Insisting the methodology be changed to suit us is not likely to succeed. It would be more productive to focus on extensive preparation for the public meeting, especially since there are opportunities for open discussion with the panelists and speakers.

There is no timeline for this Workshop available yet. Nor do we know the members of the Steering Committee. Much will depend on the evidence review conducted in advance of the meeting. The evidence based report prepared for the Gestational Diabetes meeting (pdf link) is more than 300 pages long, and distills thousands of scientific papers into recommendations for clinical practice. Will we have to worry about psychosocial research making its way into the evidence based report? Much will also depend on selection of the panelists. According to the Consensus Development Program’s website, those panelists “must not have published on or have a publicly stated opinion on the topic.” Who will those panelists be?

We may not be able to participate in this process until the public meeting, but I hope advocates will join me in monitoring how this evolves. I also hope NIH will be forthcoming about each step in the process. In my experience, the engagement between advocates and the government is more positive and productive when the advocates have access to accurate information. We have a lot to learn about this methodology, and we should adequately prepare to have the best possible influence on the process.

 

*My description of the conference steps is drawn from the general description of the Consensus Development Program on NIH’s website and an email from Dr. Susan Maier, Chair of the Trans-NIH ME/CFS Research Working Group. The actual process may differ from what I describe here.

 

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Showcase

Posted on March 19, 2013 by Jennie Spotila

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

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

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

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

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

The patient session on April 25th will be watched closely by more than just ME/CFS stakeholders. If I were an advocate for ALS or gastroparesis or narcolepsy, I would pay very close attention to this session as the test case for how future PFDD meetings will be conducted. The same is true for drug developers and researchers, including those who do not currently work on ME/CFS. This meeting has the potential to be one of the biggest stages our ME/CFS community has ever had.

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

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

We need to stop whining and complaining, and start preparing for this meeting. We have four hours on April 25th to describe how ME/CFS affects us, list the many treatments we’ve tried, and make the case for immediate investment in developing new treatments. We will have the microphone for half a day, and we will be watched by FDA, HHS, drug companies, and advocates for other diseases. What we say at the meeting will help us or hurt us. Showing drug developers and FDA that there is a market for new treatments, and telling them which symptoms to target and how to measure improvement, will help us. Complaining about the meeting or the long list of things that FDA and other agencies have done wrong will hurt us.

This meeting is a tremendous opportunity for us to be heard. Let’s give voice to the symptoms and treatments we deal with. Let’s fill the public docket with comments. Let’s carefully craft our answers to the questions FDA has posed to us. If you can attend the meeting, sign up to be a panelist or commenter. If you can’t make it, sign up for the webcast and submit written comments. I’ve explained how the signup process works and the questions FDA is focused on, and I’ll be doing more posts in the next 5 1/2 weeks. The CFIDS Association is planning some webinars to help you prepare as well.

This is our moment. FDA wants to hear what we have to say about our disease and the need for treatment. We have to get this right. Let’s use this opportunity to teach people about our disease. Let’s show the world what we need, and that we’re willing to participate constructively in the process to get it.  There’s no way to know when (or if) we’ll have this opportunity again. Let’s bring our A game to this showcase event.

 

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Quick Announcement

Posted on March 18, 2013 by Jennie Spotila

Finally (!) I am able to share the news that I’ve been invited to serve on a panel at the upcoming FDA Drug Development for ME/CFS Workshop. I’ve wanted to announce this for awhile, but panelists were asked not to publicize their participation.

I’m serving on a panel with clinicians and other patients to talk about symptoms, treatments, and possible outcome measures. The agenda and speaker list for the meeting should be released soon, and I’ll be writing more about the meeting in the coming weeks. I’m honored to be included on a panel of distinguished experts, and to have the opportunity to give voice to patients’ experiences.

More news to come . . . .

 

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Tell It To The FDA

Posted on March 15, 2013 by Jennie Spotila

The much anticipated FDA meeting on drug development for ME/CFS on April 25-26th is fast approaching, and your participation is needed! There is a great deal of time set aside for patient input, but the process will work very differently from what we’re used to at CFS Advisory Committee meetings. In this post, I will pick apart the process so you can participate in the most effective way possible.

First, if you want to attend the meeting in person you must register by April 8th. Space is limited, so sign up soon. If you want to watch the meeting via webcast, you must register. The webcast is observation only, not participatory.

Second, there are several ways to offer comment at the meeting. This is not the CFSAC format of five minute slots, one patient after another, with no interaction. The session on April 25th from 1pm to 5pm will collect patient input on the impact of ME/CFS on patients’ lives and individual experience with current treatment regimens. You can participate in several ways:

  1. Apply to be included on a patient panel addressing one or more of the questions FDA has identified in the meeting notice. My understanding is that each panel will feature approximately five patients, and each will have a minute or so to address the specific question.
  2. Participate in the facilitated discussion after each panel. This part of the session will be opened to the floor, but it will be facilitated to stay focused on the specific question.
  3. Offer comment during the open public comment session on topics not addressed by the panels. There will be time limits enforced in order to give everyone a chance to speak.
  4. If you cannot attend the meeting in person, you can submit written or electronic comments. Comments will also be accepted after the meeting through August 2, 2013. Electronic comments can be submitted to regulations.gov using the docket number FDA–2012–N–0962.

What are the questions FDA wants to address? The Federal Register notice for the meeting (pdf link) lists the following questions that patients will be asked to address during the April 25th session:

Topic 1: Disease Symptoms and Daily Impacts That Matter Most to Patients
1.  What are the most significant symptoms that you experience resulting from your condition? (Examples may include prolonged exhaustion, confusion, muscle pain, heat or cold intolerance.)
2.  What are the most negative impacts on your daily life that result from your condition and its symptoms? (Examples may include difficulty with specific activities, such as sleeping through the night.)
a) How does the condition affect your daily life on the best days and worst days?
b) What changes have you had to make in your life because of your condition?

Topic 2: Patients’ Perspectives on Current Approaches To Treating CFS and ME
1.  What treatments are you currently using to help treat your condition or its symptoms? (Examples may include FDA-approved medicines, over-the- counter products, and other therapies, including non-drug therapies such as activity limitations.)
a) What specific symptoms do your treatments address?
b) How has your treatment regimen changed over time and why?
2.  How well does your current treatment regimen treat the most significant symptoms of your disease?
a) Have these treatments improved your daily life (for example, improving your ability to do specific activities)? Please explain.
b) How well have these treatments worked for you as your condition has changed over time?
c) What are the most significant downsides of these treatments (for example, specific side effects)?

It is very important that patients address these questions through their comments. The input collected during this afternoon session will be used during the scientific meeting on the second day to inform discussion about endpoints, outcome measures, and clinical trial design.

How do I get on a panel? You must register for the meeting and apply for the panel discussion by April 8th. Indicate which topic(s) you want to address and provide a brief summary of your answers to the questions. FDA will identify panel participants and confirm with those individuals in advance of the meeting. You must physically attend the meeting to participate on a panel.

How do I get a public comment slot? You must register for the meeting and apply for the public comment session by April 8th. You will be asked to provide a brief summary of your comments via email to ME-CFS-Meeting@fda.hhs.gov. FDA will notify those with public comment slots in advance of the meeting.

Where do I submit comments? You can submit comments electronically through regulations.gov using the docket number FDA–2012–N–0962. You can submit written comments to the Division of Dockets Management (HFA–305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. Again, use the docket number FDA–2012–N–0962.

REMEMBER: If you want to present comments in person at the meeting, you must register and provide your comments by April 8th. FDA will notify those selected for the panels and open public comment slots.

 

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News That Is Mostly Not

Posted on March 13, 2013 by Jennie Spotila

This week, the CFS Advisory Committee made two announcements that I think you will want to know about. First, the spring CFSAC meeting will be May 22-23rd in Washington, DC. There is no additional information about the agenda, beyond the promise to discuss the High Priority recommendation list. I’ve written summaries of the CFSAC’s recommendations to help you select the ones you think should be highest priority.

The other announcement was the publication of a document titled HHS Ad Hoc Workgroup on ME/CFS: Agency Actions and Accomplishments 2011/2012 (pdf link). This document contains very little news, but there is significance to that even so.

As I’ve covered in detail before, the Ad Hoc Workgroup was formed by Secretary Sebelius to inventory and coordinate the Department’s efforts on ME/CFS across the agencies. At the June 13, 2012 CFSAC meeting, Assistant Secretary Dr. Howard Koh said the purpose of the Workgroup was to “put forward accomplishments and opportunities for the future that would begin new efforts and conversations, identify new activities, and try to do the best we can in a very difficult funding environment.” Dr. Nancy Lee, chair of the Workgroup, said in an July 17, 2012 email to me that “The group has been tasked to identify past agency accomplishments and efforts regarding CFS and identify new opportunities for collaboration and coordination in CFS activities. . . . An outcome of the workgroup will be a report that will be posted on the CFSAC website. Because the workgroup is not yet complete in its fact-finding efforts, and because the final report will need to be reviewed by all participating agencies, it will probably not be posted until 2013.”

So now it is 2013, and this Agency Actions and Accomplishments report was published on the CFSAC website on March 11th. What was the outcome of the Workgroup’s efforts? The bulk of the report catalogs activity in research, patient care and education. There is nothing new here. All of the activities were previously reported at the 2011 and 2012 CFSAC meetings. Were any of these activities stimulated by the Workgroup or the display of interest from the Secretary’s office that led to its creation? There’s no way to tell.

One intriguing detail was that Office of the Assistant Secretary for Planning and Evaluation participated in the Workgroup. This is something I had hoped would happen, although it was not announced before now. Of course, there is no way to tell from the report whether this individual made positive contributions to the meetings.

The significance of this report becomes evident only through careful parsing of the introductory summary. Describing what led to the creation of the Workgroup, the report says, “Given the budget environment, it was important to determine how to best leverage these programs and resources to demonstrate how agencies’ efforts address ME/CFS priorities, but also signal the Department’s commitment to addressing the needs of people living with ME/CFS.” In other words, we need to make the most of the resources we currently have, but we also have to prove that we’re committed to helping people with ME/CFS. Hence, the Workgroup was created. I suppose that one’s point of view will determine where you think the emphasis lies: in maximizing resources or in making it look like they’re doing something.

Last August, I wrote, “What we’ve been promised is a report in 2013 that inventories what is currently being done across HHS, and which will hopefully include new initiatives (possibly the national webinar and patient registry). Dr. Koh’s caution about the budgetary climate is, in my opinion, code for ‘don’t expect anything new that will cost a lot of money.'” This report, unfortunately, falls in line with my prediction.

One thing that remains unclear is whether this is the final report and the end of the Workgroup. That is certainly the expectation created last year by Drs. Koh and Lee. But the report says, “The Workgroup is a forum to discuss agency accomplishments and explore opportunities for future activities. Several agencies are investing in future programs that will specifically address ME/CFS while others have resources geared toward a wider population that could benefit people with ME/CFS and their families.” (emphasis added) The report describes itself as the Workgroup’s “first report,” but there is no specific promise or indication of what the future plans for the Workgroup may be.

I expect we will hear more about this report at the May 22-23rd CFSAC meeting. I hope HHS won’t try to spin this as a great deliverable that should excite the community – it most certainly is not. It would be much more helpful is for HHS to explain what impact, if any, the Workgroup had on fostering new activities that were not already planned, as well as whether and how the Workgroup will continue to function in some capacity.

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Research Recommendations

Posted on March 12, 2013 by Jennie Spotila

See Part One – Tangled Web
See Part Two – CFSAC Specific Recommendations
See Part Three – Care and Services Recommendations
See Part Four – Education and Training Recommendations

Appropriately enough, the CSFAC has made more recommendations on research than any other topic. See pages 1 – 7 of the Recommendations Chart (pdf link). It’s too cumbersome to review them one by one, but we can start by setting aside many as complete or obsolete and then cover the rest by category.

Finished or Obsolete

The greatest number of completed recommendations relate to CDC. Through 2009, the CSFAC made twenty-one recommendations related to CDC research, leadership and funding. Eighteen of those are marked as complete. I think this designation is accurate for several reasons, and in fact, I consider all twenty-one to be complete or obsolete. For example, I think recommendations related to the restructuring of the CDC program in November 2007 should be discarded as either extremely unlikely (e.g. place an extramural effort in the Office of the Director) or irrelevant (e.g. recommendations from the Blue Ribbon Panel). All of the recommendations related to the composition and focus of the Blue Ribbon Panel from May and October 2008 are also obsolete, as that Panel met in late 2008. Finally, all the recommendations regarding the CDC’s five year plan are obsolete as that plan has been mothballed ahead of schedule (although I would love to hear the early death of that plan explored at a CFSAC meeting).

Oddly, one of these completed recommendations was included in the CFSAC’s High Priority list. One of the five-year plan recommendations included a subpart saying that “Identification of biomarkers and etiology of CFS” should be a priority area (May 2009). I’ve previously explained how that subpart was combined with other recommendations to create a new recommendation never voted on by the Committee.

There are several other recommendations that I think should be discarded as obsolete. The recommendation for a intramural staffed laboratory (September 2004, repeated August 2005) and the recommendation for CDC and NIH to sponsor focused workshops (September 2004, repeated August 2005, marked complete by CFSAC) have been partially acted upon, and the language probably needs clarification to reflect that. Both the recommendation to include CFS in the NIH Roadmap Initiative (November 2006) and endorsement of the NIH State of the Knowledge meeting (October 2008) are accurately marked complete. Another recommendation is omitted from the Recommendations Chart, but might be considered complete in any case. The September 2004 recommendation urges the use of a Request for Applications with set aside funds. NIH did in fact issue an RFA for CFS in 2005, so it’s fair to mark this one complete.

Two recommendations marked complete on the Recommendations Chart are not complete, in my opinion, and I cover those below. In addition, one recommendation was omitted from the Chart, and one was miscategorized as Research and I covered it in the Education section. By my count, that leaves fifteen recommendations for review. These break down into recommendations on Centers of Excellence, NIH funding, and specific kinds of research.

Centers of Excellence

The CFSAC has long supported the idea of regional centers for CFS that would offer the best care, as well as opportunities for research and education. The specifics have changed over the years as the Committee has repeated and refined the idea, but they have recommended such Centers six different times. Three of them have no progress reported:

Direct the NIH to establish five Centers of Excellence within the United States that would effectively utilize state of the art knowledge concerning the diagnosis, clinical management, treatment, and clinical research of persons with CFS with funding in the range of $1.5 million per year for five years. (9/04; 8/05)

Establish Regional Centers funded by DHHS for clinical care, research, and education on CFS to provide care to this critically underserved population, educate providers, outreach to the community, and provide effective basic science, translational, and clinical research on CFS. (5/09)

Establish Regional Centers funded by DHHS for clinical care, research, and education on CFS. (10/09)

Two recommendations are presented on the Chart in abbreviated form. The May 2007 recommendation includes five paragraphs of text laying out the dire need for research and clinical care and how regional centers could begin to meet that need. The Chart includes only the bolded text from that page:

HHS establish 5 regional clinical care, research, and education centers, centers which will provide care to this critically underserved population, educate providers, outreach to the community, and provide effective basic science, translational, and clinical research on CFS. (5/07)

The October 2010 recommendation is an odder situation. Both the Chart and the CFSAC webpage for the recommendation list only two sentences:

Develop a national research and clinical network for ME/CFS (myalgic encephalomyelitis/CFS) using regional hubs to link multidisciplinary resources in expert patient care, disability assessment, educational initiatives, research and clinical trials. The network would be a resource for experts for health care policy related to ME/CFS. (10/10)

However, the minutes of the meeting indicate that the recommendation continued for several paragraphs more. The text makes the argument for why centers would address physician education, clinical care and translational research, and further recommends a national network of large databases to support sub-typing and focused treatments. (CFSAC Minutes, October 14, 2010, pp. 53-56)

The most recent recommendation in this category is from November 2011:

CFSAC would like to encourage and support the creation of the DHHS Interagency Working Group on Chronic Fatigue Syndrome and ask this group to work together to pool resources that would put into place the “Centers of Excellence” concept that has been recommended repeatedly by this advisory committee. Specifically, CFSAC encourages utilizing HHS agency programs and demonstration projects, available through the various agencies, to develop and coordinate an effort supporting innovative platforms that facilitate evaluation and treatment, research, and public and provider education. These could take the form of appropriately staffed physical locations, or be virtual networks comprising groups of qualified individuals who interact through a variety of electronic media. Outreach and availability to underserved populations, including people who do not have access to expert care, should be a priority in this effort. (11/11)

This recommendation was included in the High Priority list. It was also addressed in Assistant Secretary Dr. Howard Koh’s response to the Committee on August 3, 2012 (pdf link). Dr. Koh said that the Ad Hoc Workgroup was developing a Department-wide plan and opportunities for interagency collaboration. CFSAC recommendations would be considered by the Workgroup, and the finished plan would be posted on the CFSAC website. But readers of this blog will remember that in reality, we will not be getting a plan and the chances of funding regional centers seem very slim indeed.

 NIH Funding

If CDC-related recommendations have been accurately marked complete, the same is not true of the NIH-related recommendations. NIH is the largest source of biomedical research funding in the Unites States, and it is appropriate for it to be the focus of the CFSAC.

Only one recommendation is not expressly about the amount of funding available from NIH. At the October 2012 meeting, the Committee recommended “establishing a dedicated standing committee for ME/CFS at NIH.” The Recommendations Chart contains a note referring to the Trans-NIH ME/CFS Research Working Group, although the document referenced is not responsive to this recommendation. But the minutes of the meeting make clear that what was contemplated by the CFSAC was not a standing committee like the Trans-NIH group already in existence, but a standing study section responsible for reviewing ME/CFS related grant proposals (CFSAC Minutes, October 3, 2012, p. 44).

The same cannot be said for the other CFSAC recommendations on NIH funding:

Based on the positive response to the NIH’s Request for Applications issued in July 2005 (funded in 2006), the Committee recommends equivalent funding for a second RFA. (11/06)

CFSAC recommends to the Secretary that the NIH or other appropriate agency issue a Request for Applications (RFA) for clinical trials research on chronic fatigue syndrome/myalgic encephalomyelitis. (11/11)

Both of these recommendations are marked as complete on the Chart. The notes refer to NIH Program Announcements which do not have dedicated funds set aside. As I have said to the CFSAC in the past, a recommendation for an RFA with set aside funds cannot be considered completed through program announcements with no dedicated funding.

The last two recommendations have no progress noted on the Chart.

ME/CFS is an illness with enormous economic and human costs. The April 2011 NIH State of Knowledge Workshop identified a number of gaps in what is known about the illness. To address these gaps warrants an interagency effort comprising, but not limited to, NIH,CDC, and AHRQ. Further, the focus should be on interdisciplinary discovery and translational research involving interacting networks of clinical and basic science researchers. Areas to be examined would include the following: identification of patient subsets for detailed phenotyping and targeted therapeutic interventions, biomarker discovery, systems biology approaches and disability assessment.(5/11)

CFSAC recommends that you instruct the NIH to issue an RFA (funded at the $7-10 million range) for projects to establish outcomes measures for ME/CFS diagnosis, prognosis and treatment which would include but not be limited to biomarker discovery and validation in patients with ME/CFS. (10/12)

The May 2011 recommendation is notable for its inclusion on the High Priority list. However, as I’ve previously noted, the recommendation was substantially altered both on the Chart and on the list. Specifically, both documents delete the following text:

To facilitate the above goal, CFSAC recommends that ME/CFS research receive funding commensurate with the magnitude of the problem and that the NIH (and/or other appropriate agencies) issue an RFA specifically for ME/CFS.

If this recommendation is to be designated as a high priority, the full text must be included. That deleted sentence sets the spending level (commensurate with the problem) and the method (an RFA). These are essential elements of the recommendation.

Specific Types

Finally, CFSAC has made recommendations that focus on particular topics or kinds of research. I’ve listed each in its entirety below along with any relevant notes from the Chart.

DHHS should provide funds to develop an international Network of Collaborators that would allow for multidisciplinary CFS-related research using standardized criteria accepted by the international CFS research community. (9/04, 8/05)

The full recommendation continues, “Such a network would pool large number of patients from around the world, and would require investigators to develop and employ common protocols.” This is different from the Centers of Excellence recommendations because it specifically contemplates international collaborations and standardized criteria and common protocols.

Promote, encourage, and fund research directed toward the diagnosis, epidemiology, and treatment of CFS in children and adolescents. (9/04; 8/05)

This recommendation is marked completed and the Chart refers to several program announcements in the notes. However, this focus area is one of ongoing concern and it does not seem fair to consider it finished.

Finally, two recommendations from October 2012 focus on specific patient populations. The Chart assigns the agency responsibility to CDC, but it seems to me these are relevant to NIH as well:

CFSAC recommends that you allocate specific funds to study patients with ME/CFS from past cluster outbreaks. (10/12)

CFSAC recommends that you allocate funds to study the epidemiology of patients with severe ME/CFS. (10/12)

 Keep in Mind

Of the fifteen recommendations, six relate to the Centers of Excellence concept. Four recommendations relate to NIH RFAs, and one was intended to create a permanent committee (or study section) and NIH. The last four recommendations relate to international collaboration, pediatrics, cluster outbreaks, and severely ill patients. More than any other category, the recommendations on research have been condensed in the Chart and much of the excluded language is important. This has to be considered when selecting recommendations for the final designation as high priority.

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Education and Training Recommendations

Posted on March 11, 2013 by Jennie Spotila

See Part One – Tangled Web
See Part Two – CFSAC Specific Recommendations
See Part Three – Care and Services Recommendations

The CFSAC lists fourteen recommendations in this category, but I covered one recommendation on ICD-10-CM in the Care & Services section. See pages 8 – 11 of the Recommendations Chart (pdf link). There are also some errors in this section of the Chart. One recommendation was repeated, and another was omitted. I’ve also added a recommendation listed in the Research section of the Chart. I point out the recommendations affected by these errors below.

Health Care Providers

Training health care providers to diagnose and treat ME/CFS patients is a critical need, but none of the CFSAC’s recommendations about it were included in the High Priority list. Their recommendations have not been successful, either.

Among CFSAC’s first recommendations was to make CFS a topic of training of health care providers at regional and national conferences sponsored by the Department (September 2004, repeated August 2005). No progress is noted on the Chart. Similarly, CFSAC said the Secretary should recognize the challenges of ensuring CFS is part of any efforts to train or educate health care providers (May 2010). No progress is noted, but the wording does not actually request specific and measurable action.

CFSAC requested that the Surgeon General to send a letter to state health departments and other professional organizations informing them about the education programs and resources (May 2007). No progress is noted here, either, but an update was part of a recommendation the following year. CFSAC requested that a concept paper be produced for consideration by the Surgeon General for development of a workshop (May 2008) as a preceding step to a Surgeon General letter. This recommendation contains a lengthy preamble making the case for such a workshop. This workshop was never held, and the Chart notes no progress on this recommendation. Finally, CFSAC recommended that an AHRQ review of the science be communicated to key medical education outlets, and that a Surgeon General’s letter be disseminated to clinicians and other health professionals (October 2009). No progress is noted, and the AHRQ review was never completed.

Finally, CFSAC recommended that the CFS Toolkit for health professionals be removed from the CDC website (June 2012). The recommendation does not appear in the Chart. However, Dr. Elizabeth Unger of CDC told the CFSAC that the Toolkit would remain on the CDC website until it could be revised, despite the Committee’s recommendation otherwise. (CFSAC Minutes, October 4, 2012, p. 12)

Despite the CFSAC’s obvious concern about educating health care providers, these recommendations have been unsuccessful. CDC has undertaken the production of CME and other provider education resources, but this has proceeded separately from the CFSAC recommendations. CDC’s stubborn refusal to remove the Tool Kit is significant and disappointing.

General Public

The idea of increasing public education through a public awareness campaign was first recommended by CFSAC in September 2004 (repeated August 2005). The CDC conducted a public awareness campaign from November 2006  through 2008, and this recommendation is marked complete. CFSAC recommended that the FY08 and FY09 budgets for the CDC campaign be increased beyond the FY06 level based on the positive initial response to the campaign launch (November 2006). This recommendation is marked complete, and includes a progress note detailing the results of the campaign. Finally, CFSAC recommended that HHS develop a CFS Toolkit for patients and caregivers, similar to the Toolkit for Providers (October 2008). No progress on this recommendation is noted, and CFSAC never revisited the issue.

One puzzle about this area is that the High Priority list included the September 2004 recommendation, despite its status as complete. Until the Committee discusses the priorities at the spring 2013 meeting, we won’t know if the members believe a new public awareness effort is needed and what parameters they are considering.

Others

CFSAC has recommended education activities through a variety of channels, which would have the effect of reaching different audiences. First, CFSAC encouraged education for Social Security reviewers and adjudicators (September 2004, repeated August 2005). The Chart note says that training for SSA adjudicators is ongoing. Second, CFSAC recommended that HRSA communicate with Area Health Education Centers regarding the availability of CME programs through CDC (May 2008). This recommendation is marked complete, and the Chart notes that HRSA communicated to its networks in the summer of 2008.

CFSAC returned to the issue of disability adjudication in 2011, recommending that a workshop be organized to engage disability assessment experts in order to produce a document for patients and adjudicators which could contribute to more efficient and fair disability processes (May 2011).  This recommendation is listed twice on page 10 of the Chart, and no progress on this recommendation is reported (although a note related to the CDC Toolkit is listed there).

To help parents of children with CFS to access resources at the Department of Education, CFSAC recommended that a link be added to the CFSAC website for the Department of Education’s Parent Technical Assistance Center Network (June 2012). This recommendation has been completed. Finally, the Committee recommended that HHS should partner with CFSAC members and the Department of Education to educate teachers and school nurses on ME/CFS in children and adolescents (June 2012). The progress note states that this issue is being addressed through the HHS Ad Hoc Workgroup, including information dissemination through the Administration for Children and Families.

 Keep in Mind

Of these fourteen recommendations, four have been completed (two on the public awareness campaign, HRSA communication, and link to parent resources). The recommendation to remove the CDC Toolkit was refused. Efforts to train Social Security adjudicators and cooperate to educate school personnel are listed as ongoing. The other six recommendations have no progress noted at all.

 Go to Part Five – Research Recommendations

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