Occupy CFS

I submitted two versions of testimony to the May 22-23, 2013 CFS Advisory Committee meeting. My written testimony can be viewed here.  What follows is the testimony I delivered by telephone this morning:

My name is Jennifer Spotila, and I thank my fellow advocates for being there in my stead. My comments today differ from the written testimony I submitted last week, because there is new information that I must bring to your attention.

First, the FDA Drug Development Workshop put an exclamation point on the need for more research in order to accelerate the identification of effective treatments for this disease. Unfortunately, not only is our government failing to fund research commensurate with the burden of illness, but funding is decreasing at an alarming rate.

Last year, NIH projected that it would spend $6 million on ME/CFS in 2012. But when I totaled the spending for the grants linked to the ME/CFS category in NIH’s Estimates of Funding for Various Research, Condition and Disease Categories, I found that NIH actually spent just over $4.5 million. This is a decrease of almost $2 million or nearly 30% from 2011 spending.

Let me repeat that for emphasis. NIH funding for ME/CFS research in 2012 dropped by nearly 30%.

But it is worse than that. Three of those grants are unrelated to ME/CFS. These grants comprise 18% of the 2012 total. If we remove those grants, the total spent by NIH drops to just under $3.7 million.

Let me repeat that for emphasis. NIH funding for ME/CFS research in 2012 was just under $3.7 million.

Whether you use NIH’s total of $4.5 million, or you use my adjusted totals to remove unrelated grants, this is an unacceptable drop of nearly 30% from 2011 funding levels. Is this what the Obama Promise looks like? NIH funding for ME/CFS research is at its lowest level since 2008. Do I really need to repeat that for emphasis?

We are not alone in seeing a decline in funding from 2012. Of the 235 disease categories listed on the NIH site, close to 30% show a decrease from 2011 (although only 11 other categories saw a decrease of 20% or more). But this means that almost 70% saw an increase in funding, so I do not accept the excuse that times are tough.

Dr. Maier has said many times that there are not enough applications coming in. She is right. Approximately 20 ME/CFS applications were submitted to NIH last year. Obviously, such a low number of proposals will not get the job done. At the same time, NIH has invested set aside funds for other conditions in order to stimulate research. For example, the MAPP initiative for chronic pelvic pain that you have heard about at prior meetings started with $40 million in funding set aside by NIH. The RFA process exists to promote and encourage research into areas of need.

To be perfectly frank, I am tired of the chicken and egg debate of which comes first, the money or the larger number of proposals. Here’s a novel idea: do both at once. Advocates, organizations, and researchers should do everything in their power to encourage more proposals, and NIH should pony up dedicated funding. Try both and see what happens.

How can we be positive about change, as Dr. Jones said this morning, in light of these numbers? This is a crisis situation. I do not accept a 30% drop in funding. I do not accept plummeting backwards to 2008 funding levels.

The second issue I must bring to your attention is the High Priority Recommendations List that you will be asked to approve this afternoon. Less than 30 minutes has been allocated for this discussion, and my information is that you will be voting on the original list as drafted in January 2012. I am sorry to say that this is patently ridiculous. If you allow this, you will be approving a list that is 18 months old, that includes none of your recommendations from 2012, and that includes two recommendations that have already been completed. Furthermore, the wording of several recommendations have been altered from the original versions, and you will be making this decision without hearing any of the public comment scheduled for tomorrow.

If you go along with this, you are telling the patient community that designating high priority recommendations is a formality not worthy of meaningful discussion or even the slightest effort to produce a current, up to date list. I ask you, Committee members, to move this discussion to Thursday afternoon so that you can devote the attention and consideration that this document deserves. If you do not, then I ask you to vote NO and not approve the list in its January 2012 form. We deserve more than formalities. Thank you.

The CFS Advisory Committee will meet on Wednesday and Thursday this week (May 22nd and 23rd). The meeting will be webcast, and I’ll update with that information as soon as it becomes available. UPDATED: To call in to the CFSAC in listen only mode: 1-866-500-6250, participant code: 9487727.

UPDATED: WATCH THE MEETING LIVE

The full agenda has been posted, and I’ve put together some things you can expect and watch for at the meeting.

New Faces – You may recall that Dr. Jacqueline Rose resigned after the June 2012 meeting. Her replacement has been appointed: Rebecca Collier, RN. All we know about Ms. Collier is that she is a registered nurse, and that she nominated herself to the Committee in 2011. I’m looking forward to learning more about her at this meeting. This will also be the first meeting attended by the nonvoting liaison representatives from the CFIDS Association, IACFS/ME, and the New Jersey CFS Association. We don’t really know how the liaisons will be integrated into discussions, or how their expertise and perspectives will be considered.

Missing Faces – Dr. Ann Vincent recently resigned from the Committee for unknown reasons. Other missing faces will be those of many patients who regularly attend the CFSAC meetings. Most of the advocates I know are still recovering from the FDA meeting and are too ill to make the trip, myself included. The room may look a little empty.

Official Presence – For the past several years, Assistant Secretary for Health Dr. Howard Koh has briefly attended the CFSAC meetings to give an official welcome and review highlights of the Department’s work on ME/CFS. He is not listed on the agenda for this meeting. Instead, Principal Deputy Assistant Secretary for Health Dr. Wanda Jones will give the official welcome. Dr. Jones is well liked by patient advocates, so this is not necessarily a bad thing, but it will be concerning if Koh is stepping back from his involvement in the CFSAC.

Agency Actions & Accomplishments – In March, the HHS Ad Hoc Workgroup published its first report. There really wasn’t any news in that report, but I expect the Department will cite this as a significant accomplishment. I hope we will hear about the future plans for the Workgroup, and whether they will actually stimulate new programs for ME/CFS.

High Priority List – Dr. Nancy Lee assured Public Citizen that the High Priority List would be fully discussed at this meeting. However, the current agenda allocates only 30 minutes for the approval of both the Priority list and the proposed list of organization websites. I honestly don’t know how there can be “full discussion” of the list in less than 30 minutes. I hope that Committee members will recognize that this is insufficient time, especially since public comment may reflect the varied opinions we have about the List. My own opinion on the High Priority List is here.

NIH Funding – As I said last week, NIH funding for ME/CFS research fell by more than 20% in 2012. Dr. Susan Maier is on the agenda for Thursday, and I am eager to hear what she has to say about the drop. I hope that CFSAC members will press for an explanation and for real change.

Case Definition – Last October, CFSAC recommended that the Secretary “promptly convene (by 12/31/12 or as soon as possible thereafter) at least one stakeholders’ (ME/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.” Obviously, that meeting has not happened. We do know that NIH has started a case definition process, so I hope we will hear more about that. In addition, a group of advocates have asked DHHS to stop using the broken Fukuda definition and begin using the Canadian definition. Despite all of this activity, case definition is not on the agenda for the meeting. Has the Committee done any work on this since October? Will the Secretary authorize CFSAC to hold a case definition meeting? Its absence from the agenda does not bode well.

Q&A – For the first time, a combined 45 minutes has been designated for “Public Q&A.” This could be very interesting, because I know that advocates have dozens and dozens of questions for the CFSAC and ex officios at every meeting.  I have no information on how this will be conducted, but it seems likely that it will be similar to the way questions were collected at the FDA meeting. Audience members submitted questions on cards, and moderators selected questions to pose the panel. I heard a lot of grumbling after the FDA meeting that very few questions were answered, and that the tough questions were not selected. Advocates have said the same thing about CDC’s PCOCA calls. Will this be a similar procedure, or will we see something like “open mic”?

Time – The allocation of time on this agenda is very different from previous meetings. Public comment has been reduced by one hour, but 45 minutes of Q&A was added. Ex officio reports have been reduced by 30 minutes, and lunch increased by the same amount. There were 5 hours scheduled for presentations at the October 2012 meeting, but that has been cut almost in half for this meeting. The biggest change is the increase in Committee discussion time from 1 hour, 50 minutes in October 2012, to 4 hours, 15 minutes for this meeting. This change will be welcome if the Committee uses that time to explore issues in greater depth and formulate better recommendations.

NIH spending on ME/CFS research has been controversial since I’ve been in advocacy, largely because the funding numbers are so low compared to the burden of this disease on people and the economy. I’ve examined the details of spending in previous years, including an analysis of 2011. The 2012 numbers are now available, and the news is not good at all: funding has fallen off a cliff to its lowest level since 2008.

First, recall that NIH had projected to spend $6 million on CFS research in 2012, the same as the amount spent in 2011. Unfortunately, NIH now says that it spent $5 million in 2012, and projects the same amount for 2013 and 2014. Even worse, when I dug into the actual grants, I found that NIH had spent far less than $5 million.

There are 16 grants listed for 2012 spending (several grants appear twice on the list because they received funding from more than one Institute) for a total of $4,518,182. This is a decrease of $1,827,966 or 28.8% from the 2011 funding. In 2011, ME/CFS was 218th out of 235 disease categories funded by NIH. In 2012, we dropped to 224th place.

Unrelated Grants

To make it worse, three of the grants NIH included in the 2012 ME/CFS list are not actually related to ME/CFS:

• A grant to Dr. Matthew Hayes for $80,000 is investigating the potential mechanisms that cause nausea and malaise after the administration of a class of drugs for diabetes in the hope of developing treatments for obesity. I have no idea why this is categorized as CFS-related, unless it was the use of the word “malaise.”
• Two grants relate to XMRV, and as such should also be excluded as non-CFS related. NIH’s own study on XMRV (the Lipkin study) established that XMRV is not found in CFS patients or controls, and the original Science paper was retracted in December 2011. There is no reason to allocate XMRV spending to CFS research. Dr. Monica Roth received $293,436 as part of her ongoing study in the integration of murine retroviral vectors. Dr. Jeffrey Cohen received $448,678 in a new intramural NIH study that did not detect XMRV in CFS patients or patients with other chronic inflammatory diseases, and also tested use of a particular drug in a person with herpesvirus infection – although that patient does not appear to have CFS.

These three grants account for $822,114 or 18% of the total NIH claims to have spent on ME/CFS. Obviously, less than $1 million is not much in research terms, but for our field it is a huge amount.

Category Breakdown

After excluding the three unrelated grants, we are left with thirteen grants for a combined total of $3,696,068. Let’s see how they break down by category:

• Two studies tested psychological treatments. Dr. Fred Friedberg received $368,424 for his ongoing study of home-based illness self-management. Dr. Michael Antoni received $541,505 for his study of telephone based patient-partner cognitive behavioral stress management.
• In the area of orthostatic intolerance, Dr. Anthony Ocon received $47,232 to continue his study of nitric oxide and orthostatic intolerance in CFS patients. Dr. Dikoma Shungu received $269,536 for a treatment study of an amino acid and its impact on oxidative stress. Dr. Shungu’s grant was a new award in 2012 (and is distinct from his $2 million grant announced in March 2013).
• Two new grants will look at the role of the gut microbiome in CFS, with Dr. Maureen Hanson receiving $227,220 and Dr. Mary Ann Fletcher receiving $181,621. Both of these grants were new awards in 2012.
• The remaining grants investigate neurological, endocrine, or immune aspects of ME/CFS for a total of $2,060,530. The grants to Dr. Nancy Klimas, Dr. Theoharis Theoharides, Dr. Vincent Lombardi, Dr. Benjamin Natelson, Dr. Kathleen Light, and Dr. Leorey Saligan are all continuations of grants funded in 2011. Only one grant is new: Dr. Roland Staud will investigate the peripheral and central mechanisms of fatigue and pain through specific receptor cells in muscles in patients with ME/CFS.

So how does this stack up against prior years? Not very well.

As you can see, the 2012 spending is the lowest total amount since 2008. Orthostatic intolerance research is at its lowest point in five years, and psychological research is at its highest percentage since 2008. The only good news is that for the first time in five years, neurological/endocrine/immune research received more than 50% of the total. But I’ve made this point before: if I’m a researcher and I see 20% of such a small amount of money going to psychological research, will I invest the time and effort to submit a biomarker proposal?

Another interesting point is that four of the grants funded in 2012 were new awards (totaling $1,026,377 or 22.7% of the total), and all four of those grants were reviewed by the CFS Special Emphasis Panel. Dr. Susan Maier, chair of the Trans-NIH ME/CFS Working Group, said at the October 2012 CFS Advisory Committee meeting that 18% of ME/CFS applications were funded in 2012, and that this represented a higher success rate than average at NIH. But according to Dr. Sally Rockey, Deputy Director for Extramural Research, that is not the case. Dr. Rockey’s analysis shows that the overall success rate for research grants at NIH in 2012 was 18%. I guess we should be glad for the parity, but if 18% of grants were successful then this means that there may have been only 20 applications for ME/CFS research at NIH in 2012. I checked with several sources, and they estimated that 20 to 22 applications were submitted to NIH last year. Obviously, such a low number of proposals will not get the job done.

XMRV Effect

Another issue that I’ve investigated is the effect of XMRV funding on overall ME/CFS research expenditures. Was XMRV funding raising the overall level of research funding? Or was non-XMRV funding increasing as well? Last year I said: “Spending for 2012 is projected to be $6 million. If all of that money is actually spent on CFS studies, it will represent a significant increase over 2011 because the Lipkin study funding would be shifted to other CFS studies. That would be a significant win for CFS patients.” Unfortunately, the exact opposite happened. When you strip out the non-ME/CFS related grants (including XMRV) for funding in 2008 through 2012, you see this:

XMRV spending did inflate the spending numbers in 2009-2011, but after stripping that out we still saw an increase in funding during those years. Now we are sliding backward, and fast.

DO SOMETHING

Whether you use NIH’s raw number of $4,518,182 or my adjusted number of $3,696,068, the precipitously decline in funding remains evident. The NIH number represents a 28.8% decrease from 2011, and my adjusted number represents a 26.2% decrease from 2011. This is insane. NIH must do something to stimulate research in this area. There can be no argument about this. NIH must DO SOMETHING!

In testimony at a Senate hearing, NIH’s Principal Deputy Director Dr. Lawrence Tabak stated that NIH spent $386 million in pain research in 2011. NIH now reports that $479 million was spent on pain research in 2012, an increase of $93 million or 24%. One contributing factor for this meteoric increase was that the Affordable Care Act of 2010 included specific requirements for pain research and care. But whatever the reasons, it’s still a boatload of money. To me this reinforces the point that advocates have made over and over and over: there IS money available. It’s just being allocated to “higher priorities” than ME/CFS.

I wrote an article for Phoenix Rising about a few things we can expect at the upcoming CFS Advisory Committee meeting on May 22-23rd. Today I want to focus on why you should participate, and how to do so. This is urgent because you have less than one week to send your comments in to the CFSAC!!!

Dr. Nancy Lee has said that the Committee will be discussing the High Priority recommendations list. You might recall that an earlier version of that list was trashed after Public Citizen pointed out that it had been created behind closed doors and without public input, and therefore was in violation of the Federal Advisory Committee Act. That’s why the list is on the agenda for this meeting.

This is YOUR chance to tell the CFSAC which of their recommendations you think should be the highest priority. Their last attempt at this included recommendations on research funding but also included conducting a public awareness campaign and understanding how recommendations are sent to the Secretary. Are those your highest priorities?

We have to speak up. If we stay silent, we send a signal to the CFSAC: go ahead and make decisions behind closed doors without our input. If we stay silent, then we have no right to complain that the CFSAC priority list does not reflect our own priorities. If we stay silent, then it is likely that the final list will not reflect what we think is most important.

Do not give in to apathy! I know we are all crashed after the FDA meeting. Even the staunchest advocates are struggling to find the energy right now. But we need to do this. We need to sign up for public comments slots if we can, or at least write to the CFSAC if we don’t have energy to speak. Even if all you do is write down your top three priorities and send that in, every message they receive makes it harder for them to ignore the views of the community.

Here’s how you can make your voice heard on the highest priorities:

• Look at this list and choose between three and five of the recommendations that you feel should be made the highest priority by the CFSAC.
• Sign up for a public comment slot if you are able to speak for five minutes. The deadline is May 15th!
• Write down your highest priority recommendations and email it to CFSACMay2013@seamoncorporation.com. Do this even if you are too sick to use a speaking slot. It is very important that you send your comments in so that they become part of the public record, so make sure you send them in by the May 15th deadline!

Make your voice heard! Tell the CFSAC what you think should be the highest priorities for DHHS. Whatever the outcome, I think we’ll be stuck with the list for some time, so please do whatever you can to express your views NOW.

As I’ve reviewed elsewhere in great detail, the CFS Advisory Committee will be discussing which recommendations should be designated “high priority” at their meeting on May 22-23rd. We now have the opportunity to voice our own views about which recommendations are most important. I’ve saved you the trouble of reviewing all 77 recommendations yourself, and have selected the recommendations that are incomplete and still relevant. The full text of those 17 recommendations are listed below for your convenience. I’ve listed them in chronological order, and have added a summary sentence in bold at the beginning of each recommendation. If you want to dive into the full list in greater detail, I review all the recommendations by category starting here.

1. Establish Five Regional Centers for ME/CFS research, clinical care and education. The magnitude of CFS warrants a strong call to action to address the unmet needs of this underserved population. CFS affects at least one million Americans, 85% of whom have not been diagnosed. The degree of functional impairment experienced by CFS patients is equivalent to patients with end-stage renal failure, COPD, and late-state HIV/AIDS.
   The CFSAC has listened to public testimony at each of its meetings, and meeting after meeting patients and family members bring heart rending stories of limited access, physicians with no knowledge of diagnosis or treatment, and lives set aside waiting for effective treatment. These lives set on hold translate into an enormous economic impact, with families effected losing half of the household income, costs the U.S. economy $9.1 billion per year in lost productivity alone and up to $25 billion annually when medical costs are added to that estimate.
   The lack of diagnostic markers and effective treatments, or even treatment guidelines, represents a crisis for evaluation and management of this large population. These factors in combination mandate the creation of innovative measures to focus resources on identifying solutions to these critical problems.
   There have been basic science advances which should be leading to new treatment strategies, yet progress in translating these advances into effective treatments has been slow. This is in large part due to a complete lack of clinical care centers and research centers. Investigators are frustrated by a lack of access to representative patient populations, and patients are frustrated by a lack of accessible expert clinical treatment centers. Funding mechanisms to develop new centers for either clinical care or centers for research are shrinking, but the needs of this underserved very ill patient population are unmet and growing.
   Therefore, the CFSAC recommends that the Secretary use the resources and talent of the agencies that make up the HHS to find ways to meet these needs. One starting point is our request that the 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. The advisory committee understands that fiscal exigencies have to date prevented the formation of these previously recommended centers, but it is our hope the Secretary will use the full weight of his office to effectively fund this program through existing funding mechanisms that might be available or new programs. (5/07)
2. Establish interagency/interdepartmental effort to coordinate support for children and young adults with ME/CFS. CFSAC recognizes that much can be done to ensure that every child with CFS has the best possible access to support and treatment and asks that the Secretary facilitate a taskforce or working group to establish an ongoing interagency and interdepartmental effort to coordinate school, family, financial, and health care support for children and young adults with CFS. (10/08) This was included in the High Priority List
3. Fund CDC research into biomarkers and viral etiology. Provide adequate funding to CDC to effectively carry out a detailed 5-year plan. This should include, but not be limited to, immediate progress in these priority areas (Resubmitted from May 2009 with minor modification to [a]): a.     Identification of biomarkers, with increasing efforts in viral etiology of CFS: (10/09) This was included in the High Priority List in heavily modified form.
4. Use regional hubs to create a national research and clinical network for ME/CFS. 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)
   
5. Adopt the term “ME/CFS” across HHS programs. (10/10) This was included in the High Priority List.
6. Organize a disability workshop. CFSAC asks that HHS organize a workshop to engage experts in disability assessment, the outcome being a document useful to patients and adjudicators which could contribute to more efficient and fair disability process. (5/11)
7. NIH should fund ME/CFS research commensurate with the magnitude of the problem, and issue an RFA specifically for ME/CFS. 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. 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. (5/11) This was included in the High Priority List in modified form.
8. Pool resources to create Centers of Excellence, using physical or virtual locations. 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 was included in the High Priority List.
9. Classify ME/CFS at G93.3 in the ICD-10-CM. This multi-part recommendation pertains to classification of CFS in ICD classification systems: (a) CFSAC considers CFS to be a multi-system disease and rejects any proposal to classify CFS as a psychiatric condition in the U.S. disease classification systems.(b) CFSAC rejects the current classification of CFS in Chapter 18 of ICD-9-CM under R53.82, chronic fatigue unspecified, chronic fatigue syndrome, not otherwise specified. (c) CFSAC continues to recommend that CFS should be classified in ICD-10-CM in Chapter 6 under Diseases of the Nervous System at G93.3 in line with ICD-10, the World Health Organization, and ICD-10-CA, the Canadian Clinical Modification and in accordance with CFSAC’s recommendations of August 2005 and May 2011. CFSAC rejects CDC’s National Center for Health Statistics Option 2 and recommends that CFS remain in the same code and the same subcode as myalgic encephalomyelitis becauseCFS includes both viral and non-viral triggers. (d) CFSAC recommends that an “excludes one” be added to G93.3 for chronic fatigue, R53.82, and neurasthenia, F48.8. CFSAC recommends that these changes be made in ICD-10-CM prior to its rollout in 2013. (11/11) This was included in the High Priority List.
10. RFA for clinical trials research. 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) This was included in the High Priority List.
11. Remove the CDC Toolkit. CFSAC asks that the Centers for Disease Control and Prevention (CDC) remove the CFS Toolkit (both English and Spanish versions) from the CDC website. (6/12)
12. Educate educators and school nurses about ME/CFS. CFSAC asks that HHS partner with Committee members and the Department of Education to educate educators and school nurses on ME/CFS affecting children and adolescents. (6/12)
13. NIH should issue a $7-10 million RFA for outcomes measures, and biomarker discovery and validation. 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)
14. CFSAC recommends that you allocate specific funds to study patients with ME/CFS from past cluster outbreaks. (10/12)
15. CFSAC recommends that you allocate funds to study the epidemiology of patients with severe ME/CFS. (10/12)
16. CFSAC recommends that you endorse the Coalition4ME/CFS Option 1 proposal for the ICD-10-CM that was recommended at the September 19, 2012 NCHS public meeting. (10/12)
17. Hold a stakeholders’ workshop to reach a consensus on case definition. CFSAC recommends that you will promptly convene (by 12/31/12 or as soon as possible thereafter) at least one stakeholders’ (ME/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. (10/12)

As of today, I have been appointed to the FDA’s Patient Representative Program. This has actually been in the works for awhile, and I’ve been anxious to tell you about it, but it finally becomes official today.

The FDA Patient Representative Program selects and trains patients (or family members) to serve on advisory committees and to provide FDA with the perspectives of people affected by serious diseases. I learned of the program last October while researching information about FDA. I submitted an application on October 23, 2012, and received provisional training in December. But the conflicts of interest screening process takes months to complete, and I was not notified until March that I was officially accepted. My appointment begins today, May 6th, and will run for five years.

What does this mean? First, I am now eligible to serve on advisory committees convened by FDA to evaluate drugs or biologics for the treatment of ME/CFS. You may recall that Alaine Perry served as the patient representative on the advisory committee that reviewed the Ampligen application in December 2012. Second, FDA offers ongoing training to its patient representatives so I will have the opportunity to learn more about various issues (like risk mitigation programs, etc), and to share the perspectives of ME/CFS patients with FDA and my fellow representatives. I have already learned a great deal about FDA through this program and I look forward to learning more.

Federal ethics rules for service on advisory committees are very strict. I would be disqualified from service by even the undue appearance of a conflict of interest. I won’t be able to comment on products that may come up for review, or accept compensation from companies that make those products (of course, no one has ever offered me such compensation!).

My sincere hope is that there will be multiple opportunities in the next five years for me to serve on committees evaluating treatments for ME/CFS! In the mean time, I will pass on what I learn through this program. I also hope that you will help me educate FDA about ME/CFS by sharing your perspectives and questions with me, so that I can share those views with them.

When I uploaded my Crash Day 3 video on Monday, I was scared. In fact, I almost didn’t do it. I can’t control the fact that I have crashes, but I can control who sees me that way. Like most people, I withdraw when I feel lousy. The suffering that comes with the worst days of ME/CFS is not something that I have ever wanted to display in public. I originally recorded the video in order to submit it to the FDA comment docket. But after I did that, I thought maybe it was worth sharing with you because it might be similar to what other patients experience. I felt very vulnerable putting it out there, but it seemed like the right thing to do.

Wow. Just . . . Wow! Your response has been incredible, and far greater than I anticipated. The video has 893 views on YouTube as of today. There were 388 hits on the blog on Monday, rising to over 1,000 hits yesterday. You’ve been sharing it on Facebook, on the forums, on Co-Cure, on Twitter, and your own blogs. And your comments – here on the blog and on Facebook and by email – have been overwhelming in kindness and support.

I thank you from the bottom of my heart. It felt like I was taking a big risk, but you caught me when I took that leap of faith. You reminded me I am not alone, and you used my video to help teach others about this disease. Not only do I feel supported, but we’ve turned this crash into something meaningful and productive! So keep sharing the video and keep the conversation going. And please know that you have my deepest gratitude for the love and support you have given me this week.

On the suggestion of my friend Claudia, I recorded a video of myself on the third day after the FDA Drug Development Workshop on ME and CFS. I had to swallow my pride to do this. Even some members of my family have not seen me in a full-blown crash like this. But Claudia was right: the FDA needs to compare how patients looked at the meeting to how we look and feel afterwards. It’s not pretty. But I also know I am not the only person struggling through a crash right now, so here goes:

Here is what I looked like at the FDA Meeting on April 26th:

And here I am on the third day after the meeting:

I’m not sure which way to describe the ME/CFS community reaction to the announcement that Kim McCleary will be leaving the CFIDS Association: that people were so stunned you could hear a pin drop or that the news was the equivalent of a nuclear bomb detonation. Whatever way you describe it, the news is huge.

I worked closely with Kim during my service on the Association’s Board of Directors (2006-2011), and especially during my two years as Chairman. Kim is my friend, and my feelings about her announcement are both personal and professional. Most patients do not know how hard she has worked on their behalf. Kim dedicated herself to our cause, and has made tremendous personal sacrifices in service to that mission. I don’t think there is anyone who knows more about the politics of the disease, and few who understand the research better. In the last few years, she has been instrumental in the transformation of the CFIDS Association into a cutting edge research organization. It’s probably too soon to define her legacy, but she will forever be one of the giants in the battle against ME/CFS.

I know that some in the community will want to rehash various criticisms of Kim and decisions that she made. That’s up to them, but I will not. No one can lead an organization for twenty-two years without there being disagreements and debate. I see little value in revisting them now.

The real issue – and what I believe advocates and patients should be focused on – is where the CFIDS Association goes from here. Can the organization thrive without Kim? The answer is YES. Here’s why the CFIDS Association will continue to succeed:

• Research – The quality of the Association’s research program is unmatched. Faster Cures has included the Association in TRAIN  – an initiative that encourages innovation in research and venture philanthropy. Strategic funding decisions have led to a 7 to 1 return on investment. For every dollar Association donors have invested in research grants, those researchers have secured an additional $7 in federal grant money. This success is unparalleled. In a recent webinar, Dr. Vernon described the Association’s cutting edge and unmatched patient-centered approach to research and finding treatments for the ME/CFS.
• Vision – While Kim has been the visionary driver of the Association, the staff and Board are committed to innovation and cutting edge solutions. Suzanne Vernon is a big thinker and is passionate about the Association’s mission. She has recruited new researchers to the field, like Drs. Kathy and Alan Light, Dr. Gordon Broderick, and others. She sees the big picture of the disease, and is always looking for new and innovative ways to attack the problem. The entire staff recently completed a three-year strategic plan, and from what I have heard, Kim’s departure has required adjustments but not fundamental change to that plan. Combining that vision with the expertise of the staff and Scientific Advisory Board means that the Association will not lose step during the transition.
• Staff – Kim has been the face of the Association, but the staff are some of the unsung heroes of this movement. This is not a 9-to-5-it’s-just-a-job group of people. The staff work harder for less reward than just about anyone else I have ever known. Their dedication, expertise, and persistence has made the Association’s success possible.
• New Blood – Mark Stone and Leigh Reynolds have joined the staff within the last year, and both bring expertise from working with other disease populations. They have proven themselves in building other organizations with the support of patient communities, and they are bringing those skills to ME/CFS.
• Profile – The Association is one of the most – if not the most – visible ME/CFS organizations to the outside world. Their proposal took the top prize in the Sanofi challenge. When the XMRV story first broke in 2009, the Board said they wanted the Association to be the source of credible and current news for both the ME/CFS community and others. Both Kim and Suzanne helped raise the profile of our disease by interfacing with new policy makers and influential people, and some of those people now serve on the Association’s SAB.

Kim’s long tenure may have created the impression for some that she was the Association, but that is not the case. The staff, Board, donors, and supporters have always been the lifeblood of the organization, and that has not changed. There is no doubt that Kim’s departure will mean a time of transition for the Association, but the mission and the plan to identify disease-modifying treatment for ME/CFS will go on. Thanks to Kim, the Association is off to a very good start.

The FDA Drug Development Workshop for ME and CFS is finally here!!! I will be speaking on a panel at the meeting on Friday, and am in frantic preparation mode. I’ve gathered the pertinent materials together in one post to help you prepare as well. This is an historic meeting, and I hope you will watch and participate.

WATCH THE MEETING – I will add the link as soon as it becomes available Watch it here
Meeting Materials

FDA’s meeting materials page

Final Agenda (pdf link)

FAQs about the meeting (pdf link)

Preparing to Participate

Finding Your Strongest Voice for Public Testimony webinar by the CFIDS Association on April 4th

Training for the Drug Development Workshop webinar by PANDORA on April 16th

Understanding FDA

FDA 101 webinar offered by FDA on April 18th

Overview of the Drug Development Landscape webinar offered by the CFIDS Association on March 28th

How FDA Defines “Safe and Effective Treatment” webinar offered by the CFIDS Association on April 11th

FDA’s Drug Approval Process

Drug Approval Process Infographic (pdf link)