Care and Services Recommendations

Posted on March 8, 2013 by Jennie Spotila

See Part One – Tangled Web
See Part Two – CFSAC Specific Recommendations

Only ten recommendations are assigned to this category. See pages 12 – 15 of the Recommendations Chart (pdf link). Most relate to the categorization of CFS in the ICD-9 and ICD-10, but the others cover a range of topics. I’ve also added two recommendations, one each from the Education & Training section and the CFSAC Specific section.

Clinical Care

A smattering of recommendations touch on various aspects of clinical care or guidelines. Regarding HHS efforts, the CFSAC recommended an interagency and interdepartmental effort to coordinate support for children and young adults with CFS (October 2008). No progress on the recommendation is reported, and it was included in the High Priority list. CFSAC also recommended that CMS, AHRQ, and HRSA should develop a demonstration project focused on more efficient and effective care (May 2010), and the progress note states that more must be known about causes and treatment before such a project could be pursued.

In the midst of the XMRV issue, CFSAC recommended that CFS patients be indefinitely deferred from donating blood (May 2010). This recommendation is accurately marked complete, given the current guidance in the blood community. Finally, the CFSAC recommended that HHS make the IACFS/ME Primer widely available (June 2012). This recommendation is accurately marked complete since the Primer was posted to guidelines.gov.

Classification

The classification of CFS in the International Classification of Diseases manual (ICD) has been the subject of Committee recommendations since September 2004. This is a significant (and difficult) issue concerning how CFS and ME are coded, and those codes communicate information about the diseases. For example, ME is listed in the Neurological Disease category, but in the United States version CFS is listed in the Signs and Symptoms chapter. CFSAC members and patient advocates (particularly the Coalition 4 ME/CFS) have repeatedly recommended that CFS be moved to the neurological category. In the US, the customization of the ICD is done by the National Center for Health Statistics at CDC. The CFSAC recommendations on this issue are split between the Care & Services category and the Education category. To help this discussion, I’ve included the October 2012 recommendation from the Education category here (see page 11 of the Recommendations Chart).

CFSAC’s first recommendation was to classify CFS as a “Nervous System Disease” (September 2004, repeated August 2005). The progress note on the chart states “The science of CFS does not support this action at this time.” CFSAC revisited the issue in 2010, but there is some confusion in the record about what they intended by the recommendation. Both the Chart and the CFSAC website state that the CFSAC rejected a proposal to classify CFS as psychiatric, and that it should retain its current classification in the Signs and Symptoms section (May 2010). However, the minutes of the meeting do not support this characterization of the recommendation, stating that the Committee unanimously approved a recommendation that “CFSAC strongly rejects the proposal to classify CFS as a psychiatric condition in the ICD 10.” (CFSAC Minutes, May 10, 2010, p. 40) I do not know why the extra sentence was added to the versions reported on the Chart and website.

CFSAC recommendations became more closely aligned with proposals from the Coalition 4 ME/CFS the following year. First, the Committee recommended that CFS be classified in the nervous system chapter, and reject the proposal to retain the “CFS, unspecified” code in the Signs and Symptoms chapter (May 2011). CFSAC reiterated this position in a multi-part recommendation to classify CFS in the Diseases of the Nervous System chapter, and not the Signs and Symptoms chapter (November 2011). Progress is listed as “ongoing” and the note indicates that the recommendation was shared with the NCHS. This recommendation was included in the High Priority Recommendations list. Finally, CFSAC expressly endorsed the Coalition 4 ME/CFS Option 1 proposal recommended at the September 2012 NCHS meeting (October 2012). This proposal would move CFS to the Nervous System chapter and combine it with myalgic encephalomyelitis in a single code.

At the October 2012 meeting, Dr. Nancy Lee said “her sense is that the process is large and ongoing and she does not believe the Secretary would want to interfere in a process that deals with 10,000 diagnoses in order to weigh in on one.” (CFSAC Minutes, October 4, 2012, p. 57)  On August 24, 2012, the final implementation of the ICD-10-CM was delayed until October 1, 2014.

Name and Definition

The name and definition of our disease has been controversial for decades. The CFSAC’s predecessor committee, the CFS Coordinating Committee, formed a Name Change Workgroup but the Committee was dissolved before its work was finished. Written recommendations from the Coordinating Committee were submitted to the newly formed CFSAC, and in December 2003 the CFSAC issued a position statement saying, “we feel that a change of this name to another name should occur only when there is a better understanding of the pathophysiololgy of the illness.” Since 2003, the term “ME/CFS” has gained greater acceptance and new case definitions have been proposed. Still, the CFSAC did not return to the topic for the purpose of making recommendations until years later.

The first such recommendation was inspired by the CDC’s empiric case definition (a long summary of the definition and associated problems can be found here). CFSAC rejected the definition and the term “chronic unwellness,” and recommended that HHS commit to support a national effort to arrive at a consensus case definition (October 2009). This recommendation is found in the CFSAC Specific section of the Recommendations Chart on page 17. The Chart notes that CDC uses the 1994 Fukuda definition, and that “chronic unwellness” is not a component of the definition. Nothing more is said in the Chart about finding a consensus definition.

After Dr. Dennis Mangan said he would change the name of the Trans-NIH Working Group to include the term ME/CFS, the CFSAC recommended that the term ME/CFS be adopted across HHS programs (October 2010). This recommendation was included in the High Priority list. There has been some penetration of the name ME/CFS at NIH and CDC, but the Department has not officially responded to the recommendation.

Finally, after a lengthy and sometimes contentious discussion, CFSAC recommended that the Secretary promptly convene a stakeholders’ workshop to reach consensus on a case definition (October 2012). The progress note on the chart states that NIH is planning a workshop on the research case definition and CDC is collecting data that will be useful for case definition discussion.

Keep In Mind

Three recommendations are marked complete (blood donation, the Primer, and retain CFS in Signs and Symptoms), and that is accurate. Two recommendations have progress notes indicating no action will be taken (2004 ICD-9 and HRSA demonstration project). Ongoing progress or no progress is claimed on the remaining recommendations (interagency task force on pediatrics, 2011 and 2012 ICD-10-CM issues, name change, and case definition).

 Go to Part Four – Education and Training Recommendations

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CFSAC Specific Recommendations

Posted on March 7, 2013 by Jennie Spotila

See Part One – Tangled Web

The CFSAC Specific category contains eleven recommendations regarding the Committee’s own operation. See pages 16 – 18 of the Recommendations Chart (pdf link). However, one recommendation concerns the case definition so I will cover that in the next post. The rest are a bit of a mixed bag, but they break out into a few topics and most are listed as complete.

Ex officio Membership

The ex officio members of the CFSAC are non-voting representatives from many HHS agencies. These representatives are important to the Committee’s work because they can provide information from their agencies and offer input into discussion that shapes future recommendations. The Committee has recommended adding ex officios on two occasions. In September 2004, the Committee recommended adding representatives from Department of Defense, Department of Veterans Affairs, Agency for Healthcare and Research Quality (within HHS), and National Institute of Disability and Rehabilitation Research (within Department of Education). In November 2007, the Committee again recommended adding a representative from AHRQ.

When the Committee was rechartered in 2010, a representative was added from AHRQ and another from the Centers for Medicare & Medicaid Services. Both recommendations are listed as complete. I’m not certain, but I think that since CFSAC is an HHS advisory committee, the Secretary cannot appoint ex officios from other Departments, so I agree with the “complete” designation.

Info Sharing

Several recommendations relate to information that the Committee wanted to obtain or share (and the argument could be made that these would be better classified in the other subject areas). The Committee asked for third party insurance companies to provide information on coverage for diagnosis, treatments and rehabilitation (May 2007), but there is no action reported. The Committee also asked for reports from the agencies on the resources available for provider education (May 2007). Progress on that recommendation is listed as ongoing because ex officios give reports at each meeting, but I think it is unclear whether that fulfills the intent of the recommendation.

Anticipating the appointment of a new Secretary, the Committee requested that the transition report include information on CFS and a summary of the CFSAC’s recommendations, and the Chart lists this as completed (October 2008). Finally, the CFSAC recommended developing a list of ME/CFS organizations and criteria for posting links to the CFSAC’s website, and ongoing progress is reported (June 2012).

Position Taking

These recommendations are not really CFSAC-related, but more accurately described as positions or orientations that HHS should adopt. CFSAC recommended that HHS solicit the cooperation of the Department of Education on issues relating to pediatric CFS (October 2008). The Chart indicates that this was completed after a representative addressed the Committee in May 2009.  I disagree that this makes the recommendation complete, but the wording is so vague that it’s hard to identify the action still needed.

CFSAC also recommended that their expertise be engaged as HHS develops policy and agency responses to ME/CFS issues (October 2010). The Chart says progress is ongoing, and that members participated in planning the NIH State of the Knowledge meeting and reviewing CDC’s website content. However, the minutes of the meeting indicate that this is not the type of engagement the Committee had in mind. The intent was for CFSAC members to be participants in the process of health care reform as it related to CFS. (CFSAC Minutes, October 14, 2010, p. 56-57).

Committee Ops

Two recommendations address pure operations issues for the Committee. First, the Committee wanted to explore the possibility of adding a web-based meeting to conduct CFSAC business (May 2010). The Chart marks this as complete, noting that webinars could be used between meetings and that the public meetings are video streamed online. Holding a meeting exclusively online would still have to comply with FACA and Sunshine law requirements, so that could be challenging, and the Committee has not pursued it further.

Second, the Committee wanted to clarify the process of transmitting the recommendations to the Secretary and receiving communication back (November 2011). This recommendation was included on the High Priority list. It is marked complete, as Assistant Secretary Koh addressed it in his August 2012 letter to the Committee (pdf link).

Keep In Mind

Of these ten recommendations, most are listed as at least partially completed. The Ex Officio and Committee Ops recommendations are all complete. Regarding Info Sharing, the transition report is complete, and the list of ME/CFS organizations is progressing. The requests for reports on agency resources for provider education might be considered complete (depending on the Committee’s intent), but the request for information from third party insurers was not addressed. Finally, the recommendations on cooperation with the Department of Education and engagement of the CFSAC members as experts in HHS efforts are listed as complete, but I do not believe this is accurate. However, both of the recommendations are sufficiently vague that the original intentions are unclear and as a result, progress is hard to assess.

Go to Part Three – Care and Services Recommendations

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Tangled Web

Posted on March 6, 2013 by Jennie Spotila

The CFS Advisory Committee has hit the reset button on their High Priority Recommendations, which means we will have a chance at the next meeting to offer our input on the list. We can tell the CFSAC which of their 77 recommendations we think are most important. But where to start?

I will be providing overviews of the recommendations by category, but there are two source documents you may want to review yourself. First, the High Priority List has been removed from the CFSAC website but you can see a copy of that document here. Second, the CFSAC maintains a chart of all its recommendations (pdf link) but it is not entirely accurate, as I will explain below.

Categorical

The Recommendations Chart assigns each recommendation to one of four categories: Research; Education & Training; Care & Services; and CFSAC Specific. The Chart also notes which agencies are pertinent to each recommendation, whether any progress has been made, a description of the progress (if any,) and whether the recommendation has been completed.

  • CFSAC Specific recommendations are those that relate to the operation of the Committee itself, including ex officio membership, the process of making recommendations, and other operational issues.
  • Care & Services is the smallest category. This is a hodge podge collection of recommendations, but case definition and name predominate.
  • Education & Training recommendations cover programs to educate health care providers, educators, Social Security adjudicators, and the general public. A number of agencies are included in order to cover a diversity of distribution channels.
  • Research contains more than half of the 77 recommendations made by the CFSAC. These include recommendations on the CDC research program, NIH funding, Centers of Excellence, and other scientific issues.

Tied Up in Knots

While it makes sense to work from the Recommendations Chart in choosing our own top priorities, there are some problems with the document:

  • Two recommendations were omitted from the list completely. I don’t know why.
  • One recommendation is listed twice.
  • The recommendation text listed on the chart is not always accurate. Some of them are missing sentences from the original, and some of those missing sentences are important.
  • The designated status of “complete” is not always accurate. Some recommendations have not actually been completed. There are also recommendations that are obsolete or basically completed, but not described as such in the chart.
  • A number of the Research recommendations are related to the CDC’s Five Year Strategic Plan for the CFS program, approved in 2009. Unfortunately, that plan has been retired and nothing has taken its place (that we are aware of, anyway). I don’t recall the CFSAC discussing the mothballing of the plan, so I do not know if the CFSAC believes the related recommendations are also obsolete.
  • Some recommendations are simply not actionable as written. For example, one recommendation from October 2008 reads, “[CFSAC] Endorses the planned State of the Knowledge Conference to be developed by NIH.” That’s a statement of opinion, not an action item. I’ve been critical of the CFSAC in the past for their occasional failure to make recommendations that are specific, actionable and measurable.
  • The five recommendations related to the classification of CFS in the ICD-9 and ICD-10 are split between the Education category and the Care & Services category. I’ll consider them together in Care & Services.
  • The three recommendations related to the case definition are split between the CFSAC Specific and Care & Services categories. Again, I’ll consider them together in Care & Services.

Diving In

I’ve spent the past few days puzzling over the best way to cover all this information. What do you need to know and what is the best way to communicate that to you? Let’s face it: this is dry stuff. My goal is to give you the tools you need to identify the recommendations that you think should be designated as High Priority.

Despite all the flaws in the Recommendations Chart document, it is the best starting point we have. In the posts that follow, I will summarize the recommendations in each category. I will also point out which ones are complete (or obsolete) and which ones have language missing. I will try to stay neutral in these summaries so that you can form your own opinions. In a final post, I will share which recommendations I think would make the best High Priorities.

So grab the Recommendations Chart (pdf link) and a cup of coffee, and let’s get started.

Go to Part Two – CFSAC Specific Recommendations

 

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No News is No News

Posted on March 4, 2013 by Jennie Spotila

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.

 

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Swift Response

Posted on March 1, 2013 by Jennie Spotila

I am pleased to share with you the response from HHS to Public Citizen’s letter to the Acting General Counsel about the CFSAC High Priority Recommendations. Public Citizen wrote to the Acting General Counsel on my behalf on February 14th, protesting the way CFSAC created and used the High Priority Recommendations in violation of FACA and its own charter.

On February 22, Dr. Nancy Lee responded to Public Citizen on behalf of the Acting General Counsel with this letter (pdf link):

Michael A. Carome, M.D.
Public Citizen’s Health Research Group

Dear Dr. Carome,

On behalf of Mr. William B. Schultz, Acting General Counsel and Dr. Howard K. Koh, Assistant Secretary for Health, Department of Health and Human Services, I am writing to thank you for your letter of February 14, 2013. As you noted in the letter, the document posted on the CFSAC website titled “High Priority Recommendations from CFSAC” has not been discussed or approved by the full Chronic Fatigue Syndrome Advisory Committee (CFSAC) at a public meeting. Our highest priority is to ensure that CFSAC operates openly and in compliance with the Federal Advisory Committee Act (FACA) and CFSAC’s charter.

We plan to present the document at the 2013 spring CFSAC meeting for full discussion. The document has been removed from the CFSAC website.

Sincerely,

Nancy C. Lee, M.D.
Designated Federal Officer – CFSAC

As I had hoped, involving Public Citizen in this matter resulted in swift and complete correction by HHS. Based on my previous experience sending letters to CFSAC and HHS, I expected it would take months to get any sort of reply if I had written to CFSAC about this myself. As is usually the case, a letter from an organization to the General Counsel resulted in resolution of the matter in less than two weeks. Again, I extend my thanks to Public Citizen for their help.

We now have a chance to offer input on this set of priority recommendations. The document will be presented at the spring CFSAC meeting for full discussion. Through public comment, we have the opportunity to express our opinions about the document. What recommendations do you think should have the highest priority designation?

CFSAC has made more than 70 recommendations over the past nine years. But most recommendations fall into categories like research and education. Some recommendations are obsolete or completed. Between now and the spring CFSAC meeting (which has not been scheduled, to my knowledge), I will offer summaries of these categories so that you can more easily identify the recommendations you think are most important. I suspect most of us will agree on the general issues, and I would love to see general discussion and coordination among advocates in advance of this meeting. Let’s leave no doubt in the CFSAC’s mind about what the public thinks their highest priorities should be.

 

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Coverage

Posted on February 25, 2013 by Jennie Spotila

Thank you, everyone, for your comments on my Public Citizen story. I’ve heard from you on the blog, email, Facebook and Twitter. Thanks!

We are still awaiting a formal response from the Acting General Counsel of HHS. I will share updates as we progress.

There has been coverage on several other outlets as well:

I will try to keep the list up to date. If you write about the story on your website or blog, or see it somewhere on the interwebs, please post a comment so we can track it!

Last updated March 2, 2013.

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A Public Citizen

Posted on February 18, 2013 by Jennie Spotila

When I wrote about the CFS Advisory Committee’s creation of a High Priority Recommendations document and how their process violated their own charter, many people asked me “What can we do about it?” Today, I can finally tell you what I did.

I contacted Public Citizen, a non-profit organization with the mission of serving “as the people’s voice in the nation’s capital.” Public Citizen has litigated cases under the Federal Advisory Committee Act, and has a Health Research Group that advocates for safer drugs and medical devices, equitable access to health care, and other issues.

On February 14th, Public Citizen sent a letter on my behalf to William B. Schulz, Acting General Counsel of the Department of Health and Human Services (you can read the full letter here). The letter details what I uncovered about the High Priority document, and asks the General Counsel to do two things:

We ask that you act to ensure that CFSAC complies with FACA and with its own charter going forward. We also ask that you return the January 2012 “High Priority Recommendations” document to the full committee for review, so that whatever action CFSAC chooses to take regarding those recommendations or any other transmission to HHS be debated and approved by the full committee, in public, as required by FACA.

It remains to be seen whether the General Counsel will act, and what he will do. There has been no official response as of today. However, upon checking the CFSAC website, I found that the High Priority document has been removed from the website. Perhaps this is a first step in correcting the Committee’s error.

I extend my sincere thanks to the people at Public Citizen who moved so quickly to assist me in this way. Sometimes, I feel like no one outside the CFS world cares about what we’re dealing with, and that requests for help will be met with disinterest or active disbelief. But Public Citizen did not dismiss my concerns or our disease. They recognized the public interest in the proper operation of advisory committees, and moved to help us. I am grateful for their assistance and support.

So what can you do? Help me boost the signal!!! Share my blog post, circulate it on Facebook and Twitter, and write about the Public Citizen letter on your own blogs. Tell your support groups and share it with all your contacts in the CFS world. Let’s ensure the community is informed about this action, and let’s monitor the progress together.

 

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Ampligen is Not AZT

Posted on February 11, 2013 by Jennie Spotila

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.

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On the Shelf

Posted on February 7, 2013 by Jennie Spotila

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?

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Highest Priority, Part Two

Posted on January 30, 2013 by Jennie Spotila

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.

 

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