Occupy CFS

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.

What is there to say about something like the Boston Marathon bombing? There is no sane way to reconcile the gruesome images, the suffering and destruction with our need to believe that we are safe. We are privileged enough in this country to think that this sort of thing does not happen here. Once I confirmed that everyone I love in Boston was ok, my real-world connection to the event faded. But this morning, my mind and heart are still bound up in the news.

Being housebound makes me vulnerable in an odd way. On the one hand, I am remarkably safe in my home and neighborhood. I rarely go in to the city or to places with large crowds. But on the other hand, I am an automatic audience for big news events.  I don’t have a meeting to go to, or kids to pick up from school, or any other demands on my attention. Most of the time, it’s just me, the tv, Facebook and Twitter. If I choose to tune in, I end up vicariously experiencing these events.

I watched the Columbine shootings unfold. For a week after 9/11, I only turned off the tv to sleep. Six years ago today, I followed the Virginia Tech shootings on CNN, and I held my breath between the phone calls from my brother who was there. It has gotten to the point where I start to cry as soon as I learn about an incident like these. Sometimes I have the self-discipline to avoid the media if I think the news will be too upsetting. I stayed off Twitter and Facebook for days after the Newtown shootings, and I still have not seen any footage from that day. I long ago decided that tv news is shallow, and frequently borderline moronic, so it’s easy to avoid that. But if I pay attention – and Twitter makes that incredibly easy – I get sucked in to following every update and rumor.

Emotional shock and distress quickly induces a cascade of physical exhaustion, pain, and brain fog. The more I watch, the worse I feel. Compounding the simple stress is an overwhelming feeling of powerlessness. I am trapped in my home, alone with the images of people who need help. There is nothing I can do. I remember the despair I felt after 9/11 because I couldn’t simply drive to New York and pitch in. After the Virginia Tech shootings, my brother started volunteering for an emergency services provider. I am limited to donating to the Red Cross or knitting afghan squares. This is not a substitute for directly helping people face to face. Watching people suffer, and being unable to do anything about it, feels like sandpaper against my heart.

This morning, I’m thinking about the people who will now join the ranks of the disabled. There are reports of people who lost legs or feet. Sure, they’ll get prosthetic limbs and rehab and lots of attention. But their lives will never be the same. Perhaps there will be people disfigured by the blasts, or people who develop post-traumatic stress disorder. Some of them may have great family support and health insurance, but others may not. In the moment of the explosions, everything changed for these people. They have to run a different marathon now. This is the marathon of doctors and procedures and medication and paperwork and learning to live with a changed body. This is the marathon of people helping in the immediate aftermath and then fading away and going back to their lives. This is the marathon of answering the “how are you?” and “it could have been worse” comments. This is the marathon of the sick and injured.

I don’t know what it’s like to be the victim of any crime, let alone a crime like this. But I do know about suffering and endurance and navigating a changed life. The real crisis is not the moment of explosion. It’s everything that comes after. I know a little bit about that marathon, and I wish I could help other people on that path. But my own marathon keeps me imprisoned, acutely aware that others are suffering and completely unable to help them.

Two weeks ago, NPR published a story about the rise in Social Security disability claims. The bottom line of the story is that unemployed people are choosing to go on SSDI because they have conditions that prevent them from doing physical labor and are not educated or qualified enough for desk jobs. According to this story, Social Security “has also become a de facto welfare program for people without a lot of education or job skills.” This is a legitimate issue, but there was plenty to object to in the story and I fired off an email to NPR. Since then, criticism of the story has skyrocketed, and NPR’s minor revisions of the story have not stemmed the tide. Others have done great analyses of the story and its many errors, and a coalition of disability rights advocates sent a terrific letter to NPR (pdf link).  Personally, I’m focused on a few comments by reporter Chana Joffe-Walt and the stereotypes those comments reinforce.

Joffe-Walt says that disability with Medicare could be a better deal than a minimum wage job with no healthcare. In the original version of the story, she said “it’s a deal 14 million Americans have chosen for themselves.” NPR has since revised the online text to read, “it’s a deal 14 million Americans have signed up for.” This is a distinction without a difference. Whether you use the verb “choose” or “sign up,” this is a statement that everyone on SSDI is there voluntarily. It’s the stereotype that disabled people are lazy slackers happy to live on the government dime.

I have a problem with that. I was forced to apply for, and now collect, Social Security disability. This was not anything that I chose. I got sick, I could not work, I applied for disability. And I hate collecting disability benefits. I would much rather be working, even if I couldn’t go back to the career I originally trained for and pursued. Furthermore, Joffe-Walt makes it sound like it’s easy to get SSDI. I don’t know if she actually investigated application and approval rates, but SSDI is not easy to obtain. I was denied twice, and finally succeeded after an ALJ hearing. The process took three years to complete. I know people who have spent even longer in the disability application process, and many have no other source of income during the long fight.

Joffe-Walt also points out, “Once people go onto disability, they almost never go back to work.” Gee, I don’t know, maybe because they’re disabled? Social Security requires that an applicant be disabled for at least a year, and unlikely to return to work, in order to qualify. Furthermore, the rigorous application and review process, and the years-long process of denial and appeal, would weed out all those able to return to work within a year or two of the illness or injury. Those of us who end up collecting benefits likely suffer from permanent or near-permanent disability, meaning that we will never return to work no matter how much we desire to do so.

The economics of disability is a worthwhile and complex subject. But of all the issues NPR could have covered – the cost of lost productivity, the plight of disabled people who cannot get benefits, the people who continue to work while being disabled, employment discrimination, the challenges of having invisible disabilities – of all these issues, NPR chose to focus on the slacker angle. The they’re-technically-disabled-but-have-chosen-to-exploit-the-system angle. Really, NPR? It’s like covering the economic impact of Hurricane Sandy by reporting on people who pad their losses in order to collect more insurance. Yes, it happens, but the most significant issues are more complex than the tired stereotype of people trying to collect money they do not deserve.

After the American Academy of Family Practitioners published an article on CFS in October 2012, Dr. Lucinda Bateman and I submitted a Letter to the Editor. Today, that letter was published and the full version is free online. There is also a reply from Dr. Yancey, one of the authors of the original article.

My original analysis of the article covers the methodology and the overemphasis, in my opinion, of psychological factors in CFS. In his reply, Dr. Yancey cites the PACE trial as providing evidence that CBT and GET provide “moderate benefit” to people with CFS. But since the PACE trial was published in 2011, numerous authors have documented a multitude of problems with the study design and data analysis, including this one from the CFIDS Association (pdf link).

I fear that the PACE trial will continue to dog our steps until better data are published. This is why the FDA meeting is so important, and why you should participate to the fullest extent of your ability.

Edited to add: Links to some other analyses of the PACE trial can be found here.

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.

The ME/CFS advocacy community has been hopping recently, with participation in the FDA meeting on Ampligen, my effort with Public Citizen, and Bob Miller’s hunger strike. We’ve been anticipating some sort of response from HHS, especially in the wake of Bob Miller ending his strike. Yesterday, a February 22, 2013 letter from Secretary Sebelius to Senator Reid was released but there is not much news in it. Let’s go point by point:

a few HHS employees have come forward to share their stories with me, and I share their frustration that neither a clear cause nor a cure have been identified despite some 30 years of scientific pursuit.

I’ve been hearing rumors about this meeting of patients with Secretary Sebelius for awhile. I don’t know when this meeting happened, or who the HHS employees are, but this is a good step and it’s nice to have it confirmed.

As you likely know, the Food and Drug Administration (FDA) recently convened a formal advisory group meeting to provide recommendations on the safety and efficacy of the drug Ampligen for the treatment of CFS. . . A CR letter described all of the specific deficiencies that the agency has identified in an application, allowing the company an opportunity to correct those clearly defined deficiencies in a re-submission . . . . I understand the frustration and pain of ME/CFS patients and their caregivers and how important it is that we continue to work toward development of treatments.

No news there.

FDA planned a series of activities to explore the burden of disease that impacts the quality of life for ME/CFS patients . . . During the past five months FDA hosted a teleconference and a webinar with ME/CFS patients and advocates. Additionally, the agency will hold a drug development workshop this spring to explore what is needed to facilitate development of safe and effective treatments for ME/CFS.

Again, nothing new. I summarized the September teleconference in this post and the November webinar in this one. The spring drug development workshop was announced in July 2012, and has been scheduled for April 25-26, 2013.

Other, ongoing cross-Departmental efforts take a comprehensive approach to addressing ME/CFS. The Chronic Fatigue Syndrome Advisory Committee (CFSAC) is an advisory committee that meets twice a year . . . I also established an Ad Hoc Workgroup on ME/CFS in February 2012 to increase and better coordinate the efforts of individual HHS components related to ME/CFS.

I’ve covered both of these efforts extensively. Assistant Secretary Dr. Howard Koh has been providing updates on the Ad Hoc Workgroup (which is chaired by Dr. Nancy Lee) at each of the recent CFSAC meetings. The most recent update was in Dr. Koh’s November 2, 2012 response to the CFSAC (pdf link).

The National Institutes of Health (NIH) is funding research activities that may help patients with ME/CFS. NIH is taking action to stimulate all facets of ME/CFS clinical research including clinical trials at the NIH Clinical Center with the help of its Trans-NIH ME/CFS Research Working Group. The agency . . . has also implemented a process to review applications proposing to use biological samples obtained from ME/CFS patients who participated in a recent NIH-funded study.

Dr. Susan Maier of NIH reports on the Trans-NIH ME/CFS Research Working Group at each CFSAC meeting. The opportunity for clinical trials at the NIH Clinical Care Center was announced on December 14, 2012. I covered the opportunity to use samples from the Lipkin study on September 22, 2012.

The Centers for Disease Control and Prevention (CDC) is in the final stages of a seven-site study of the clinical characteristics of ME/CFS. This study was launched in September 2011 to collect standardized data from clinical practices of clinicians with expertise in ME/CFS. The data will be used to evaluate variation in the illness among clinics, to characterize all domains of illness in ME/CFS patients, and to provide data that could be used in evaluation of a research case definition and diagnostic criteria.

This study has been the topic of much discussion at CFSAC meetings and in the patient community since it was announced, but there is nothing new here.

The Agency for Healthcare Research and Quality provided technical support to the International Association for CFS/ME for the development of a primer for clinical practitioners that has been added to the National Guideline Clearinghouse. . . The Substance Abuse and Mental Health Services Administration and the Health Resources and Services Administration are hosting webinars to educate providers in their networks about ME/CFS.

Both of these efforts were discussed at the October 2012 CFSAC meeting.

I’m not knocking this letter, nor am I knocking the advocacy efforts that led to it. It is vitally important that ME/CFS issues be on Secretary Sebelius’s radar – and Congress’s radar too. It is very possible that many of the updates in this letter were news to Senator Reid, depending on how extensively he was briefed before reaching out to Secretary Sebelius. And it’s always good to have these things in writing and on the record. This is, after all, how Washington works: communication between the legislative and executive branches give us a clue into what is (and is not) being done.

It is a little disappointing, though, that there is nothing new to us in the letter. Rumors and expectations and hopes for a stronger and renewed effort from HHS on ME/CFS have been swirling around the last few months. Did President Obama raise the priority level of ME/CFS research, as we heard last summer? Would HHS participate in the April FDA meeting in a new way? Many patients engaged in an intense email and phone campaign during Bob Miller’s hunger strike, and when the hunger strike ended Bob asked patients to stop the campaign because they had been heard.

The question is: did hearing us translate into anything new? The answer embedded in this letter is: “not yet.”

I believe that progress will be made in baby steps, rather than a big bang. We need to have realistic expectations. But we also need to see what’s in front of us. We were hoping for a new commitment or enhanced effort. This letter offers neither. I hope that concrete improvements and involvement are forthcoming, and that this letter is not just another in the long train of Free Turkeys handed out to the patient community.

In recent weeks, some ME/CFS advocates have been calling for NIH to conduct a clinical trial of Ampligen based on the reasoning that NIH conducted trials of AZT during the early years of the AIDS crisis. Unfortunately, this analogy does not hold up to scrutiny and should be abandoned as an argument in favor of Ampligen.

AZT was invented in 1964 by researcher Jerome Horwitz under an NIH grant to the Michigan Cancer Foundation. The compound was shelved after it failed early trials in mice. By 1984, when the hunt for an AIDS treatment was in full swing, AZT was the property of Burroughs Wellcome (now GlaxoSmithKline). Burroughs had done some research into AZT, and thought it might act upon HIV. However, Burroughs did not have the facilities needed to work with active HIV. At this same time, National Cancer Institute researcher Sam Broder and collaborators had developed a CD4+ cell line (the kind of immune cells vulnerable to HIV). Broder was begging pharmaceutical companies to send him compounds to test for effectiveness against HIV in that cell line. Burroughs sent a number of compounds to Broder for testing, including AZT.

In February 1985, Broder reported to Burroughs that AZT was very effective against HIV. NIH and Burroughs entered into a collaboration for the first Phase 1 trial of AZT in humans. Specifically, 19 AIDS patients were treated with the drug at the NIH Clinical Care Center in July 1985. Fifteen of those patients showed clinical improvement during the treatment. Although the drug had significant side effects, the phase 1 trial did establish that it was safe to give to humans (the objective of phase 1 trials). Conducting the remaining clinical trials of AZT was the responsibility of Burroughs. After a double-blind placebo controlled trial in 282 AIDS patients in 1986, the FDA approved AZT for use in 50,000 severely ill AIDS patients in March of 1987.

In contrast, Ampligen has been developed by Hemispherix Biopharma for more than 20 years, from inception through Phase 3 clinical trials. FDA has reviewed and denied approval to Ampligen twice (2009 and 2013). It strains credulity to claim that NIH should initiate an Ampligen study when the drug has been denied twice, particularly given that the FDA Advisory Committee voted 9 to 4 that there was not substantial evidence of efficacy. The situation is further complicated by the rumors that Hemispherix is in serious financial trouble (although its CEO was just given a $250,000 bonus). If NIH were to intervene now, that might look like another government bailout of a failing company and that hardly seems like a precedent NIH would want to set.

Coincidentally, Dr. Anthony Fauci (Director of the National Institute for Allergy and Infectious Diseases) described NIH’s involvement in drug development in an interview last week (the interview did not mention ME/CFS). Fauci said that NIH is usually involved in funding basic research and industry funds drug development and testing. Sometimes, industry conducts basic research and sometimes, NIH participates in early drug development as it did for AZT. But while each side might depart from its normal participation to a degree, he did not cite any examples where NIH first stepped in at the very end of a drug development process.

Is there any path forward for Ampligen at NIH? The CFIDS Association recently urged Hemispherix to initiate applications to NIH through the National Center for Accelerating Translational Science and the December 2012 RFA from the NIH Clinical Care Center. Whether Hemispherix will initiate such applications, and whether NIH will fund them, remains to be seen.

In the meantime, we look foolish (at best) if we claim that the Ampligen situation is just like AZT and that therefore NIH should conduct a clinical trial. The historical parallels just are not there.

I recently read Spillover by David Quammen, a book about human diseases that originate in animals. ME/CFS is not mentioned in the book, but a passage about the origin of HIV struck very close to home.

Quammen describes how HIV originated in primates in Africa in the very early 20th century. Researchers used tissue samples from the 1950s in the Congo to trace its early spread. Quammen visited the University of Kinshasa, Democratic Republic of the Congo, where those samples had been stored. In the absence of freezers and other equipment, samples were preserved in paraffin. Quammen describes the process of preservation: a tissue sample from a lymph node or other organ is dipped in successive baths of methanol, drawing out the water from the sample. The last bath is xylol to draw out the methanol. The dessicated specimen is then encased in paraffin to preserve it for storage. Quammen describes the storage area:

At the far end of the lab was another doorway, this one hung with a blue curtain. Professor Kabongo pushed the curtain aside and I followed him into a specimen pantry, narrow and tight, lined with shelves and cabinets along one side. The shelves and cabinets contained thousands of dusty paraffin blocks and old microscope slides. The paraffin blocks were in stacks and cartons, some of the cartons dated and some not. It appeared to be organized chaos. Spillover by David Quammen, p. 411-412.

The image of thousands of samples piled here and there, forgotten and left to collect dust in a hospital of a poor country, reminded me of ME/CFS.  One million patients: ignored, forgotten, and left to moulder on a shelf until one day – hopefully – a researcher comes looking for clues. That’s how I feel as an ME/CFS patient. I’ve gone through successive baths of major setbacks and treatment attempts that failed, and each one leached more life out of me. Instead of water removed by methanol, it is life and hope that is drawn out of patients, over and over and over. At some point, most of us shift from expecting to recover to simply waiting. We are like those paraffin-encased samples: inert, out of sight, unimportant. I see the room of dusty shelves of one million patients waiting for something to change. Each sample is a life frozen in time, enduring year upon year of exile.

I think this is what drives some of us to take drastic measures. We lash out at the government, we contemplate suicide, we scream and stamp our feet and wave our arms. We cannot accept personal and scientific abandonment. We will do ANYTHING to get off that dusty shelf. It is hard to understand the fury if you have never been left on a shelf to wither and die, but it is also true that fury can overwhelm and carry us away. We don’t always manage to target our efforts strategically. It looks crazy. Sometimes it IS crazy.

But ME/CFS patients have to choose: Will I endure a silent existence on a forgotten shelf, gathering dust until I die? Or will I channel the spark of rage and knock the shelf down?

When I posted the other day about the CFS Advisory Committee’s list of High Priority Recommendations (pdf link), I said that I had done some digging and that what I found wasn’t pretty. To be blunt, what I found is that the secret process used to create this priority list violated established Federal procedure, including the CFSAC’s own Charter and Bylaws. By acting in this manner, the CFSAC has disenfranchised the very stakeholders they exist to serve: the patients. And if the CFSAC continues to act this way, then CFS patients will be deprived of the one venue we have to observe and shape HHS policy on our disease.

Making Sausage

Two sources close to the process have confirmed that the subcommittees generated the priorities for this document. Selecting from the Committee’s recommendations over the years, the Education/Patient Care/Quality of Life Subcommittee and the Research Subcommittee generated a short list of recommendations that they felt were highest priority. I have not been able to get copies of those subcommittee lists, so I don’t know whether their lists were combined into the final document without further editing.

But there’s a problem. At some stage in the process, either in subcommittee or upon final compilation of the document, recommendations were altered. The fourth recommendation in the document covers the issue of research funding, and here is how it appears in the High Priority document:

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 accomplish this, specific issues would include:
a. Fund specific research for identification of biomarkers and etiology of CFS
b. NIH or other appropriate agency should issue a Request for Applications (RFA) for specific clinical trials research on chronic fatigue syndrome/myalgic encephalomyelitis. (5/11)

So it looks like this recommendation with two sub-parts was made in May 2011, right? Nope. This is an amalgam of three separate recommendations made at three separate meetings, and a sentence was deleted from one of them. Sub-part (a) has actually never been passed by the Committee with those precise words, but seems to be an edited version of a recommendation passed in October 2009. Sub-part (b) was passed by the Committee in November 2011. But the main part of this recommendation differs significantly from the version actually passed by the Committee in May 2011. The original recommendation ends with this sentence:

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.

That deleted sentence is critically important to the whole recommendation. It’s a statement by CFSAC that research funding must match the magnitude of the disease burden and that NIH should issue an RFA. The altered version that appears in the High Priority document says nothing about commensurate funding. It is a new recommendation created by smashing three recommendations from three different meetings together, deleting a critical sentence, and dating it all as coming from May 2011. I don’t know who did this. Was it done by one of the subcommittees? Was it done by Dr. Nancy Lee or other HHS personnel? There is no way to know, because this all happened in secrecy.

Behind Closed Doors

As a Federal advisory committee, the CFSAC can only make recommendations at open public meetings (barring a few exceptions not relevant here). The law requires that advisory committees discuss their recommendations in public, that they hear public comment on the issues, and then vote on recommendations in public. That is the whole point of the Federal Advisory Committee Act. But the CFSAC did not follow that procedure in creating the High Priority document.

Selecting the highest priority recommendations to forward to the Secretary is an issue that should have been discussed in public. The public should have had an opportunity to provide input on which recommendations we think are highest priority. We should have been able to listen to the committee deliberate and discuss why one recommendation should be selected over another. And there is no doubt in my mind that the entire committee should have voted on this High Priority list in public. This is especially true since the recommendation I examined above was materially altered and then combined with two other recommendations.

In fact, the process used to create the High Priority document violated both the Bylaws and Charter of the CFSAC. The CFSAC Bylaws state:

The advice of a subcommittee shall be reported to the full committee.  The full committee shall review reports and any recommendations made by the subcommittees.  Findings will be discussed at a public meeting of the full committee, at which time the full committee will determine appropriate action.

But we know that this did not happen. The advice of the subcommittees, in the form of the recommendations they designated as high priority, was simply compiled into the final document. And what happened next violated the CFSAC Charter:

The established subcommittees shall provide advice and/or make recommendations to the parent Committee. The subcommittees may not report its findings directly to any Federal official unless there is specific statutory authority for such reporting. (emphasis added)

But this is precisely what happened. The subcommittees selected their recommendations, the document was compiled, and then it was shared with Assistant Secretary Dr. Koh. Recall that Dr. Nancy Lee told me in her email of January 24, 2013, “CFSAC leadership discussed this priority list with Dr. Koh last year; he was very supportive.”

Who Cares?

Here is the problem: no one told us. The subcommittees made lists in secret. The lists were combined in secret. The final document was discussed with Dr. Koh in secret. This document was not discussed in public by Dr. Lee or CFSAC members, and it was not released to the public until this month – an entire year after it was created. In my opinion, this violates the Federal Advisory Committee Act, as well as the CFSAC charter and bylaws.

Why does this matter? Because the CFSAC is being held out as the main place we can offer input into HHS policy about CFS. In his letter to advocates on September 11, 2012, Dr. Koh said, “CFSAC provides a mechanism to ensure stakeholders are engaged and have opportunities to offer input.” Patients are not being included in other CFS-related policy efforts like the HHS Ad Hoc Working Group. We are constantly being told that CFSAC is the venue where we can offer our input.

But shenanigans like these – where the committee works behind closed doors and communicates recommendations to the Assistant Secretary without public discussion, public input or public vote – deprive us of our ability to participate in or observe the formulation of recommendations to the Secretary. If this trend continues, if CFSAC continues to do substantive work without bringing it back to the full committee in public, then we are effectively disenfranchised. We cannot allow that to happen without a fight.