How To Represent

Last month, I had my first chance to serve on an FDA Advisory Committee as a Patient Representative. I had a lot to learn about the drug under consideration, but I also learned how to use my individual perspective to represent a large group of people. As more researchers in ME try to engage with patients as partners, I have five lessons to help you use your personal ME experience to speak for us.

I have been a member of FDA’s Patient Representative program since May of 2013. FDA recognizes that patient perspectives are critical to evaluating new drugs and products, and includes patients on its advisory committees. The Patient Representative program trains the representatives and matches them with committees. Despite being in the program for more than five years, this was the first time my experience matched an Advisory Committee topic.

The Advisory Committee met on June 26, 2018 to consider a new opioid product called Remoxy. Remoxy is a new form of extended release oxycodone in a sticky, viscous gel in a sealed capsule. Pain Therapeutics (the drug sponsor) was seeking FDA approval of Remoxy with a label stating that the drug had abuse-deterrent properties for injection, snorting, and smoking routes of abuse. Since extended release oxycodone is already marketed, the Advisory Committee’s focus was primarily on the abuse deterrence claims, as opposed to the efficacy and safety of the drug itself. I was selected to be the patient representative because I have chronic pain (from ME) and have been on long-term opioid treatment.

Representation Lesson #1: Invest in preparation

Committee members received more than 300 pages of briefing materials a few weeks prior to the meeting, so I spent most of June in a crash course on abuse-deterrent properties in opioids. There was technical information about testing for abuse deterrence in the lab and clinical trials. FDA included data on the opioid abuse problem from multiple sources, which helped me understand the landscape of opioid misuse and abuse. I also did my own research on the opioid abuse crisis.

Despite the fact that I have been taking an opioid for more than ten years, I discovered that I knew very little about the crisis or abuse deterrence. I thought more than 40,000 people were dying from prescription opioid overdoses a year. I also thought abuse-deterrent opioids were proven to deter abuse. I learned that I was wrong on both counts.

The reality is that while more than 40,000 deaths a year are attributed to opioids, less than 15,000 involved prescription opioids. That is still awful, but this changed my perception of the problem and solutions. Both heroin and synthetic opioids (like fentanyl) were involved in more than 15,000 deaths apiece. Solving the opioid crisis will require solutions to the heroin and fentanyl problem, not just prescription drugs.

I was very surprised to learn that there is little evidence that “abuse-deterrent” opioids actually deter abuse. One reason for the lack of evidence is that drug companies have not submitted data to FDA from postmarketing epidemiology studies to see if there is meaningful reduction in abuse, despite being required by the FDA to conduct the studies. Another problem is that everyone (including FDA) acknowledges that there is no way to deter the most common route of abuse: swallowing pills.

Taking the time to prepare the briefing materials and do additional reading was essential. I needed to correct some of my misconceptions of the opioid abuse crisis. I also needed to understand the specific details about Remoxy and what proof there was (or wasn’t) of abuse deterrence. If I had not put in the effort, I would have been much less effective as a representative of the people for whom the drug was intended.

Representation Lesson #2: Think outside your personal box

I have my own personal experience of chronic pain and opioid treatment. After all, this is what qualified me to serve on this committee. But my personal experience is just that: individual. I needed to think beyond that.

Given my proximity to major medical centers, I have had little difficulty in accessing a pain management program. But this is not the norm. The vast majority of people with severe chronic pain are never referred to pain specialists and never receive comprehensive care.

I have also had minimal difficulty in obtaining my pain medication. We’ve made numerous changes to my medications over the last fifteen years (at least), including the permanent addition of a relatively mild opioid more than ten years ago. I don’t like taking the frequent drug tests as required by my doctor, but it’s more of a hassle than a true barrier. Again, this is not the norm.

A huge piece of the government’s response to the opioid crisis has been to crack down on the prescription of opioids. People with chronic pain are paying the price. I have read many stories of people who were told that they could no longer have the opioids they need. Even people with terminal cancer have difficulty getting adequate pain management, in part because attitudes and prescription controls have swung so far against the use of opioids.

Reading about treatment of chronic pain and the barriers people face helped me think about the value of adding another prescription opioid to the system. I came into the committee meeting thinking about balancing the needs of people with chronic pain and the needs of people who misuse or abuse pain medications. If I just thought about my own experience, I would have unintentionally introduced a great deal of bias into my perspective.

Representation Lesson #3: Identify questions in advance

When I was in law school (a long time ago!), the true challenge in preparing for an exam was the task of synthesizing all the information in order to answer questions about it. I found the same to be true in preparing for the committee meeting. Once I finished reading the briefing materials and the additional articles I found, the true challenge was identifying the questions that would help me decide whether I thought Remoxy should be approved. I knew that I had more questions than I would ever have a chance to ask in the meeting, but coming up with the list helped me synthesize everything I had read.

In the Patient Representative program training, we were encouraged to think about questions that people using a product might have. This goes beyond side effects to questions like the experience of using the product or other considerations people might have. FDA told us that we are included in meetings for the purpose of bring these kinds of concerns to the table, and that other experts might not even think to raise such questions.

In this case, I thought about how people with chronic pain make decisions about medications. Since so many of us have comorbid medical conditions and take additional medications, I had a lot of questions about the extent to which this was studied by the drug sponsor.

Since Remoxy is an opioid with potentially abuse-deterrent properties, I also wanted to know more about how a potential opioid abuser might approach the drug. How would they try to hack abuse-deterrent properties? The drug sponsor said that in addition to experts in the abuse field, they had also relied on information from abusers in designing the studies but I was disappointed to hear that they gleaned it from internet forums. Several committee members who interact extensively with opioid abusers were able to contribute important perspectives on this point.

Identifying questions in advance also helped me to be a more active listener during discussion. I reviewed my list a few times to confirm if I thought the issues had been addressed, and this helped me discern which of my remaining questions were most important to actually ask.

Representation Lesson #4: Have confidence in your knowledge and experience

It is very easy to feel intimidated when we step outside our familiar comfort zone. I am primarily housebound by ME, so it was a little disorienting to be in public, sitting at a large table while microphones and cameras recorded everything. And I knew that everyone else at the table was a doctor and/or scientist and/or advocate with professional expertise in the topic of the meeting. I also knew that I have expertise that is unique. I have relied on opioids as part of my pain management program for more than a decade, and I was sitting at the table for that very reason.

No one who spoke at the meeting (including public comment) self-identified as having chronic pain. I was surprised by this, and I felt obligated to make sure that perspective was heard. I pointed out that there are two public health crises that deserve attention: chronic pain and opioid abuse. I said there were many voices that needed to be heard at the meeting. I shared a little bit of my experience with pain management and how my healthcare is being criminalized through new restrictions. (I’ll update this post with my exact comments when the transcript is available in a few weeks)

Our society awards advanced degrees for academic work, not life experience. But if you are successfully living with a chronic disease or disability, then you have the life equivalent of a Ph.D., and it is every bit as valuable as the academic version. Being a doctor doesn’t mean understanding what it is like to be a patient. Only we can do that.

Representation Lesson #5: Be honest

Serving as a patient representative in any context requires many forms of honesty. Be honest in sharing your own personal experiences (including whether it is your experience or someone else’s). Be intellectually honest in considering other points of view. Be honest about whether you understand something. Be honest in your decision-making.

Participating on a federal advisory committee requires an open mind, especially when there are regulatory and business interests at stake. It’s a bit like serving on a jury: you need to listen to both sides before making up your mind.

The committee voted on whether the drug should be approved. This was not a secret ballot; I knew my personal vote was a matter of public record. Sharing the reasons for the vote was optional, although everyone on the committee did offer remarks. Ultimately, my vote came down to weighing all the information and making the best decision that I could.

Being honest is essential as a patient representative in every context. We have to speak honestly about the diverse experiences of people with ME, and we need to listen with an honest ear as well. I don’t think we can contribute positively without both forms of honesty.

To recap, there are five lessons from my Patient Representative experience that can help you to better represent your group:

  • Invest time in preparing the material
  • Think outside your personal box
  • Identify questions in advance, and review during the meeting
  • Have confidence in your knowledge and experience
  • Be honest in speaking and in listening

Doing those five things will help you speak on behalf of the diverse experiences of people with ME or any other group you may represent.

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NIH Forms New Working Group on ME/CFS

NIH recently announced that it is forming a new Working Group on ME/CFS. The announcement is very low on specifics, but this Working Group has the potential to make a big impact on research at NIH.

Dr. Walter Koroshetz, Director of the National Institute for Neurological Diseases and Stroke, publicly announced the Working Group at the May 24, 2018 meeting of the NINDS Advisory Council. In introducing the topic, Dr. Koroshetz asked how many Council members were conversant in ME/CFS and only two people raised their hands (37:55 of this video). That pretty much makes the case for this Working Group right there, in my opinion.

Dr. Koroshetz went on to describe the disease, the need for research, and the Collaborative Research Centers. He explained that the Trans-NIH ME/CFS Working Group is internal to NIH, and the question is how to get input from outside the NIH. Dr. Francis Collins reportedly asked NINDS to form a ME/CFS Working Group of its Advisory Council, with the goal of getting guidance from other federal agencies, and the scientific and patient communities, on how best to advance research on ME/CFS at NIH. Dr. Koroshetz said that Dr. Steven Roberds of the Tuberous Sclerosis Alliance had agreed to lead the Working Group, and that it would report back to the NINDS Council.

At the CFS Advisory Committee meeting on June 20, 2018, Dr. Vicky Whittemore made the same announcement as part of her report to the Committee. She added a little more detail, stating that members of the Working Group would be invited by Dr. Koroshetz, and that more information and a timeline would be announced in the near future. Dr. Whittemore said the Working Group would be looking at the research needs, including whether targeted RFAs are needed.

I reached out to the chair, Dr. Steven Roberds, and asked if he could share any information about the group’s plans, objectives, and membership. Dr. Roberds replied, “Because we are still in the planning stages of the Working Group, I unfortunately don’t have any plans I can share with you at this time for your article. . . I hope we have some specific information to share in the not-too-distant future.”

The membership composition of the Working Group will be key in accomplishing its goals, and the details of that charge and its timeline are of critical importance here. Will this Working Group participate in the planning of NIH’s 2019 meeting on Accelerating Research on ME/CFS? Will the Group make specific recommendations on funding amounts and mechanisms? How much public involvement will there be? We will have to see.

Dr. Roberds is the Chief Scientific Officer of the Tuberous Sclerosis Alliance. That disease has very little in common with ME. Tuberous sclerosis is a genetic disorder that causes non-malignant tumors all over the body. There are also neurological effects, specifically seizures, developmental delay, intellectual disability and autism. Only 50,000 people in the United States have the disease, and many are able to work and live healthy lives.

In stark contrast, there are an estimated 1 million people in the US with ME. While severity varies, the diagnostic criteria for the disease require a significant impact on function and an estimated 25% of people with ME are housebound or bedridden. The neurological effects are quite different from tuberous sclerosis, and people with ME experience severe effects on many other body systems, including the immune, autonomic, and metabolic systems.

So why would Dr. Roberds be selected to lead the ME/CFS Working Group for the NINDS Council? It turns out that he has significant experience in stakeholder engagement.

In 2015, Dr. Roberds helped lead an NIH meeting to update the research plan for tuberous sclerosis. The meeting was co-sponsored by NIH and the TS Alliance, and led to a set of very specific short and long-term research goals.  In 2017, the TS Alliance led an external Patient-Focused Drug Development meeting, and submitted a Voice of the Patient report to FDA.

While Dr. Roberds is not an expert in ME, he does have experience in bringing federal agencies and stakeholders together. As a member of the NINDS Council, he also has insight into NINDS, its strategic and program priorities, and the types of resources available to researchers.

This ME/CFS Working Group could be a key step forward for us. The caveat is whether Dr. Koroshetz with invite the researchers, clinicians, and people affected by ME who can make substantial contributions to the Group’s recommendations, and whether NIH will listen to those recommendations.

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Public Comment on Engaging People with ME

I delivered these comments via telephone today at the CFS Advisory Committee meeting:

My name is Jennifer Spotila. I will soon begin my 25th year of living with ME. People with ME know this disease. We have valuable insights and perspectives, not only about what it is like to live in an ME-afflicted body, but about the scientific and medical issues in this field. This is one reason why I am so encouraged that this field is beginning to engage people with ME as partners, because we can teach you.

I have had unique opportunities to learn how to integrate people affected by this disease into research and policy-making. When I served on the Board of the CFIDS Association, I reviewed grant proposals for strategic merit. I am an Ambassador for the Patient-Centered Outcomes Research Institute. I participate in the FDA’s Patient Representative Program. I served on this Committee’s Stakeholder Workgroup. Last year, I co-authored the paper Engaging People with ME as Partners in the Collaborative Research Centers, which is featured on the Faster Cures website as well as MEAction’s.

I list these qualifications to provide you with context for my comments today.

NIH and CDC are just beginning to engage us as partners. The NIH Common Data Elements project for ME/CFS included people directly affected by ME on each of its working groups, a first for the CDE program. CDC has collected input from stakeholders for its website revisions, although the focus group format is not as substantive as it could be. And for the first time in our field, NIH’s RFA for the Collaborative Research Centers required that applicants have a plan for outreach and partnering with ME/CFS stakeholders.

These are steps in the right direction, but I have questions relevant to this Committee’s advisory role:

  • Funding for the Collaborative Research Centers and Data Management Center began nine months ago, and work is underway. But not a single center appears to have a functioning community advisory board. What is NIH going to do to ensure that the Centers follow through?
  • Will NIH require stakeholder engagement in all of its ME/CFS grants moving forward? What about NIH’s intramural research?
  • What efforts are CDC and NIH making to ensure a diversity of views? Reliance upon one or two organizations for the pool of stakeholders is certainly easy and convenient for the agencies and the Research Centers, but no single organization can provide the full breadth of experiences and views that are needed in each engagement effort.
  • What will NIH do to engage people with ME in planning the April 2019 meeting on accelerating ME/CFS research?
  • How will NIH ensure that people with ME will be substantively involved in all of its own advisory groups and committees on ME/CFS research, including the newly announced NINDS Council’s ME/CFS Working Group?

CFSAC must do more than ask these kinds of questions of the federal agencies. In January 2017, this Committee received a report from its own Stakeholder Workgroup, and voted for the Workgroup to continue developing recommendations. But as far as I can tell, nothing happened. The Workgroup seems to have fizzled. As a result, this Committee has not made a single recommendation to the Secretary on how HHS agencies can effectively partner with people with ME at every stage of research and policy making. This is a significant lost opportunity.

Every person with ME is a fount of knowledge that should not be ignored. Partner with us and we will make your research and policy efforts more relevant, more accurate, and more successful.

But we are not window dressing. Engaging with us is not a box to be ticked. It is a scientific and ethical imperative that people with ME be engaged as true partners and equals with everyone else on your team or committee. Anything less than that it unacceptable.

I urge this Committee to restart the efforts of the Stakeholder Workgroup, and help lead the way towards fully integrating people with ME into both research and policy making. And I urge you to ask tough questions of the federal agencies to better understand how they will engage with us as well. Thank you.

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NIH Time Warp

Let’s take a quiz. I have a letter that was sent on behalf of Dr. Francis Collins, Director of NIH, to a person with ME, and I want you to guess when that letter was sent. Here’s an excerpt:

First, let me express my heartfelt concern for you and the millions of other men and women suffering from ME/CFS. This is a terrible disease and we must all work together, doing what each of us can, to find safe and effective treatments for ME/CFS and restore lives to health.

When do you think that was written? Ok, here’s another quote from the same letter:

It is through the receipt of meritorious research proposals that funding can increase.

Have I given it away? Here’s the last quote:

In early September, Dr. Collins reiterated NIH’s commitment to accelerating biomedical research focused on ME/CFS.

Ok, pencils down and all that. When did NIH send that letter?

Think carefully about your answer. I did give you a clue by saying it was sent on behalf of Dr. Collins, who became Director of NIH in 2009. Do you have a date in mind?

October 28, 2010. That’s right. The NIH sent this letter in 2010.

I asked you to guess the date because that letter could have been written yesterday. Very little has changed. NIH still professes to care deeply about people with ME. NIH is still pursing the strategy of waiting for research proposals to increase, rather than taking sufficient and meaningful action to stimulate research.

This letter was sent to Cheryl M. after she wrote to Dr. Collins to plead with him for more research funding. Cheryl’s letter and NIH’s response basically speak for themselves, so I have reproduced them in full here with only a few footnotes.

When she wrote to Dr. Collins, Cheryl had been bedridden for three years. Now, almost eight years later, she is still confined to a bed or recliner. Cheryl cannot leave her home more than a couple hours at a time, every few weeks. She has now been sick for 18 years.

NIH and Dr. Collins: you are brilliant at expressing sympathy. You are also brilliant at making it look like you are doing everything possible to help us. You are brilliant at telling us to be patient, and promising that you are going to fix this.

I think everyone with ME will join me in saying: It is time to fix this. Be brilliant at funding ME research. Be brilliant at finding creative ways to jump start this field, because it has languished for decades without the funding and serious attention that is desperately needed. Be brilliant at this, because Cheryl’s life is wasting away, along with at least one million others.

Here is Cheryl’s letter:

Dr. Collins,

Thank you for your participation in the recent conference on XMRV. It has filled me with much hope that our illness is being brought to light and hopefully treatments are on the horizon.

I have been disabled for 10 years due to CFS/ME and related illnesses. Due to misdiagnosis and taking a long time for an accurate diagnosis, my health deteriorated to the point of having to give up all the things that I previously did and enjoyed.

I was a very active woman before getting sick. I worked with special needs children and families, developed early childhood curriculum, was a workshop presenter/speaker for peers, volunteered heavily at my church and in the community.*

Now I am a drain on my families [sic] (parents included) finances and financial security. My illness cost my husband his job and he took a much lesser paying one to be closer to home and helping me more.

I have been primarily bedridden for 3 years. I long for the simple things in life like having the energy to walk around my block, go to church, go out with friends. Those days are gone but not forgotten.

I urge you to please fund XMRV/CFS/ME research generously. I pray for the day that I can continue an active life, as do all of the others that I know who are affected severely by this illness. So many have died by their own hands because they were too tired to keep on fighting. Please be a part of our solution. Please hear our cries for help!

Sincerely,

Cheryl M.

And here is NIH’s reply to Cheryl:

Dr. Francis Collins, Director, National Institutes of Health, has asked me to respond to your inquiry regarding National Institutes of Health (NIH)-supported research on Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS).

First, let me express my heartfelt concern for you and the millions of other men and women suffering from ME/CFS. This is a terrible disease and we must all work together, doing what each of us can, to find safe and effective treatments for ME/CFS and restore lives to health.

In early September, Dr. Collins reiterated NIH’s commitment to accelerating biomedical research focused on ME/CFS. It is through the receipt of meritorious research proposals that funding can increase. In FY 2010, the NIH provided approximately $6 million for ME/CFS and XMRV research** grants, contracts, and other types of awards to universities, hospitals and research organizations, including the Whittemore Peterson Institute. An additional $5 million was awarded to investigators studying aspects of ME/CFS, such as pain, neurological complications, genetics, blood pressure, retrovirology, and sensory processing.***

NIH is currently soliciting ME/CFS research applications through two Program Announcements. To help investigators prepare applications for research funding, NIH holds grant-writing workshops and assigns program officials to answer questions and assist with their application submissions. Dr. Collins recently asked the National Institute of Allergy and Infectious Diseases, one of the 27 Institutes and Centers at the NIH, to study a possible connection between XMRV infection and ME/CFS by using blood samples from patients diagnosed with ME/CFS.

The Trans-NIH ME/CFS Research Working Group (WG) is composed of experienced program officials highly committed to helping advance NIH supported research on ME/CFS at universities, hospitals and research institutions. The WG members serve as conduits to their respective NIH Institutes or Centers to facilitate communication between scientists and NIH leadership. The WG is hosting a major State of the Knowledge Workshop in 2011 to bring together scientists and clinicians to evaluate current ME/CFS data, propose priority areas that need further attention and then identify mechanisms for supporting such critical research.

Additionally, the NIH works with many Federal agencies and ME/CFS advocacy groups through the Department of Health and Human Services Chronic Fatigue Syndrome Advisory Committee. It is also expected that by cooperating in planning interdisciplinary initiatives, the NIH and its partners will increase support for multidisciplinary and interdisciplinary research with the goal of finding treatments and, hopefully, a cure as rapidly as possible.

Thank you again for your interest in the NIH efforts to increase ME/CFS research. We at NIH fully support your assertion that ME/CFS patients deserve the very best scientific information we can possibly produce.

Sincerely Yours,

Dennis F. Mangan, Ph.D.

Co-Chair, Trans-NIH ME/CFS Working Group

 

*I wish we knew what Cheryl could have been contributing to her profession and community if she had not been sick for the last 18 years.

**Of that $6 million, 29% went to XMRV and 6.5% was not related to ME/CFS, which means that in 2010 NIH actually spent $4.2 million on ME/CFS research. In FY 2011 (which had already started when this letter was written), NIH spent $4.6 million. And in FY 2012? $3.6 million. So funding was going down at the same time these sympathy statements were sent to Cheryl!

***I have no idea which grants this $5 million includes, but NIH has a pattern of claiming that research tangentially related to ME/CFS is directly applicable. This helps inflate NIH’s numbers but there is no way to fact check the statement in this letter.

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Talk To FDA About Chronic Pain

The FDA wants to hear from people with chronic pain, and is hosting a meeting on July 9, 2018 to collect public input. I have chronic pain, and I know that many of you do as well. This meeting is worth your time.

Management of chronic pain was not the focus of much medical or scientific attention until the 1960s. While prescription practices changed in the 1980s and 1990s with the advent of synthetic opioids, including oxycontin, it wasn’t until the beginning of this decade that pain research and policy became the focus of heavy federal attention.

Recently, that attention seems to have shifted to the opioid crisis, sometimes at the expense of people with chronic pain. There is collective emphasis on data points such as more than 40,000 people died of opioid overdose in 2016. But I had to look pretty hard to find out that only one-third of those overdoses involved a prescription opioid, and many involved another drug as well. It is not in the least bit surprising to me that patients are suffering because the current climate has made it hard, if not impossible, for people in pain to receive appropriate and effective treatment.

So this FDA meeting is very timely, and I hope it will attract significant participation. FDA wants to hear from people with any kind of chronic pain. The two main topics for discussion are:

  1. Symptoms and daily impacts of chronic pain that matter most to patients, including the experiences of pain and how it affects daily life
  2. Patients’ perspectives on current approaches to treatment of chronic pain, including barriers to accessing treatments

You can watch the meeting online and submit input to the public docket. The full details are in this Federal Register notice, but you can register for the webcast of the meeting here and submit comments here.

The meeting is part of FDA’s Patient-Focused Drug Development Initiative, and these meetings are well moderated. FDA also produces a complete transcript and a Voice of the Patient summary of each meeting.

Given the stakes for people with chronic pain, and the difficulties we continue to have in finding effective treatments, this meeting is an important opportunity to speak directly to FDA about what matters most to you.

 

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The NIH Pilot Program: What We Can Do

Credit: Wilder/Metzger

As I discussed in Part One of the NIH Pilot Program, NIH thinks that the three (and only three) Collaborative Research Centers are seeds to generate interest in ME research, and will wait for more research applications to come in over time. NIH’s slow approach to case definition is going to take ten years (or more) to show results.

I have gotten a lot of feedback asking what we can do, or expressing despair that NIH will not move fast enough to help us. There are people with ME who have lost careers, families, financial support, and more, and when I come out with a post like that one, they lose hope.

But we have to face reality in order to demand that NIH face reality and act with urgency. Don’t lose hope; get mad. If you feel despair that there are still no treatments and NIH is just twiddling its thumbs and whistling innocently, then get MAD.

We are NOT powerless!!!!! Not by a long shot. I have ideas about what we can do, collectively and individually:

Self-care: First and foremost, take care of yourself. We are stranded on a desert island, and the first thing we need to do is address our survival. Do that first. Take care of yourself, and each other. Then if you have the capacity, spend some energy on action.

Congress: When Congress expresses an interest, then NIH responds by doing something. Even the back and forth in the budget report shows that NIH will address concerns raised by Congress. So we need to keep pressing Congress – not just for report language, but the Senate resolution, and the request for a hearing. Every single person who can should try to establish a relationship with his/her/their elected officials. When those officials ask what NIH is currently doing about ME research, do what one commenter suggested and give the officials my previous post.

Dr. Collins: We can’t stop with the letter to Dr. Collins. The ONLY reason we have the CRCs and the Clinical Center study is the pressure we have applied up until now. The National Academy of Medicine report and P2P report (which both came out of attention from the CFS Advisory Committee), #MillionsMissing, and media coverage generated enough questions and enough embarrassment that Collins had to act. So we need to INCREASE that pressure. Call, email, or fax your story to Dr. Collins. Sign petitions. Are you up for direct action protests? Talk to MEAction and #MillionsMissing.

State Efforts: The advocacy community has never had the human resources to tackle state awareness issues on a broad scale before. But there is a lot you can do at the state level, and it can have an effect federally too. For example, you could urge your state health department to start tracking ME. Local efforts in New York State led to that Department of Health publishing an excellent new page about ME.  Awareness events with health department personnel or state legislators are key as well. A lot of interest and media coverage has been generated by local screenings of Unrest. Not only will you help improve awareness and care at the local level, but as more states pay attention it will put pressure on the federal agencies as well.

Researchers: I’m not letting anybody off the hook. People with ME and their allies are pushing from the advocacy side. But researchers have to push from the science side. NIH would have dismissed the science capacity of this field even more vigorously if there had been only five CRC applications, instead of ten. But what would NIH have thought if there had been fifteen or twenty applications?????? I know first hand that writing grant proposals is a time- and soul-sucking process. Researchers, do it anyway. The number of applications matters a lot. Please help us help you.

CFS Advisory Committee: There is another meeting of the CFS Advisory Committee at the end of June. Pay attention. Offer written or spoken public comment. The recommendations from that Committee do have an impact. Let’s help the Committee make stronger recommendations.

General Awareness: Tell your story. Participate in a direct action. Recruit one new person to advocacy this year. Send emails. Use Twitter! You can talk to Dr. Collins (or at least his office) by tweeting @NIHDirector. There are so many ways that you–yes, you, reading this right now–can do something to put more pressure on NIH. A groundswell will force NIH to do more than just wait five or ten years before doing something more. We are just getting started.

NIH may be content to throw seeds in the air and see which ones grow. NIH may be content to wait another five or ten years before taking an affirmative step. About one year ago, I predicted NIH’s approach:

NIH’s current strategic and policy approach to ME: risk as little as possible, cautiously drip in a little more funding, and wait patiently for something to change, some day, far down the road.

NIH wants to wait five more years or ten more years to do something different, but I’ll be damned if I wait without trying to kick up the pace. I will do all that I can to require NIH to do much much more on ME research, or I’ll die trying.

And that sure beats just waiting to die.

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The NIH Pilot Program: Wait and See

Credit: Wilder/Metzger

MEAction has collected thousands of signatures on a letter to Dr. Francis Collins, asking for a meeting with him and a plan of action to attack the ME public health crisis. That request is all the more urgent because NIH already has a de facto plan for ME, and more than a few people with ME will die before that plan produces meaningful results.

What is the plan? NIH is going to wait for the new Collaborative Research Centers to lead to more research proposals, rather than investing money to stimulate that research now. NIH has no intention of addressing the need for a consensus case definition now, and has chosen an approach that could take a decade or more.

NIH doesn’t officially call it a plan, and has neither announced it to the ME community nor engaged with us in formulating it. Denise Lopez-Majano found the plan buried in the hundreds of pages NIH submitted to Congress with its 2019 budget request. But despite how feeble NIH’s plan is, we can do something about this!

Today, I will tell you what we know about the plan. In my next post, I will share ideas for what we can do about this. NIH may be content to twiddle its thumbs and wait, but I am not.

Finding the Plan

Each year, NIH submits a request to Congress as part of the President’s budget for the next fiscal year. Congress passes a budget and includes comments back to NIH in its appropriations report. Those comments are not binding on NIH, but they do show NIH what Congress cares about. So the following year, NIH responds to that report language to show Congress how they are addressing those issues. This back and forth between NIH and Congress is not something most people pay attention to, although ME advocates have succeeded in getting favorable report language many times over the decades.

Denise Lopez-Majano found NIH’s plan for ME on pages 76 and 77 of the FY2019 document called “Significant Items.” In the report language with the 2018 budget (finally passed in March), Congress had recommended that NIH “consider increasing research funding to be commensurate with disease burden,” and suggested several types of projects to accelerate progress in the field. In its 2019 response titled “Action taken or to be taken,” NIH described its current approach to ME and deflected many of Congress’s suggestions. And between the lines of its response, NIH revealed what it plans to do (or not do) beyond FY2019.

Congress Says One Thing, NIH Does Another

RFAs: The 2018 Congressional appropriations report suggested RFAs for biomarkers and treatment trials. We already know that NIH is not doing this, and there is no plan to do it in the future.

Funding: The report suggested additional funding for investigator-initiated studies and early stage investigator awards. NIH said it encourages new grant applications, funded one fellowship, and it expects the three Collaborative Research Centers and Data Management and Coordinating Center to attract more researchers to the field. Of course, NIH failed to point out to Congress that there is no current Program Announcement for ME/CFS, which would normally signal NIH’s interest in those types of applications.

NIH also claims that 2017 funding will reflect an increase due to the CRCs as well as “new and on-going investigator initiated projects.” My analysis shows that this is not entirely true. Funding did increase overall, but without the CRCs the 2017 funding would have DECREASED by almost 15%. The only new grants were the fellowship and Dr. Kathleen Light’s grant, totaling less than $375,000 in 2017.

Case Definition: The report also suggested NIH undertake “an initiative to reach consensus on the case definition.” NIH said no. Specifically, NIH said, “Developing better diagnostics and ways to characterize patients are necessary before reaching consensus on a case definition” (emphasis added).

In other words, NIH will take no immediate action on reaching a consensus case definition. Congress suggested it. Advocates have demanded it. The P2P report recommended it. Nope! NIH wants “better diagnostics and ways to characterize patients” first. Never mind that NIH’s willful neglect is a major contributing factor to our lack of better diagnostics.

NIH Has A Plan: Do A Little And Wait

NIH’s responses in this report reveal its true approach to ME for 2019 and beyond. NIH is running a pilot program, and waiting to see what turns up. That’s the sum total of the plan, and this means people with ME are in for a very very long wait.

Yes, NIH funded three CRCs and the DMCC in 2017. But NIH could have funded more. In fact, Cort Johnson reported that, “Vicky Whittemore said the proposals were strong and if enough funding had been provided, the NIH could have funded several more centers.”

Think about that for a minute. There were other proposals good enough to be approved if there was more funding available. We could have more CRCs right now. Why don’t we? Because NIH would not invest the money.

Do you know why? Dr. Walter Koroshetz actually told us why on June 9, 2017 when he said:

I think the goal was that this was not the solution but the seed to grow the research that could then come in through regular mechanisms or else to kind of add on centers so we can kind of move the field across. I think we could fund three, three or four centers, that’s not gonna do it. (emphasis added)

NIH is planting seeds, my friends. Seeds. And NIH is content to wait and watch those seeds grow. I admit that I am not much of a gardener, but I do know that you can’t just wait on seeds. You need to prepare the soil, water frequently, and fertilize. THAT is how you help those seeds grow into a fruitful garden.

There is another piece of NIH’s slow strategy: the intramural Clinical Center study led by Dr. Avindra Nath. This study is using very tight selection criteria and is doing the deepest dive ever into the pathophysiology of ME. The results from this study will inform–but not conclude–NIH’s direction on those better diagnostics and patient characterization that NIH sees as the prerequisite to establishing consensus on a case definition.

Credit: Wilder/Metzger

So how long will this take?

First, it’s important to remember that the Clinical Center study is small. As Dr. Nath said in March 2016, “[Y]ou don’t want to do a huge expansive study the first time around. If it takes 1000 patients to find something, it probably isn’t worth chasing a result.”

The study was originally planned to have 40 ME/CFS patients, 20 healthy controls, 20 post-Lyme patients, and 20 people with functional movement disorder. The movement disorder group was subsequently dropped and the healthy control cohort increased to 40.

From the very beginning, Dr. Nath cautioned that the study would take awhile, with each subject requiring a week at the Clinical Care Center, and an additional two weeks for those who come back for the second visit. The first visit was later extended to two weeks for people with ME because they cannot undergo as many tests in a day as the other groups. Dr. Nath kindly provided me with an update on the number of subjects who have gone through the protocol and those still remaining as of May 7, 2018:

  • People with ME (first visit, 2 weeks): 13 of 40 completed
  • People with ME (second visit, 2 weeks): 2 of 20 completed
  • Healthy Controls (first visit, 1 week): 16 of 40 completed
  • Healthy Controls (second visit, 2 weeks): 5 of 20 completed
  • Post-Lyme Controls (first visit, 1 week): 0 of 20 completed
  • Post-Lyme Controls (second visit): 0 planned

Based on the number of weeks per visit, the 36 total visits so far should have taken 56 weeks. The reality is that it has been 82 weeks since the first subject visited NIH (Dr. Nath previously said visits began at the end of October 2016). No doubt some of the delay is due to holidays and other calendar issues, and some of it was caused by working out the kinks in the protocol. However, even if all delays were eliminated, there are still 164 weeks remaining to complete data collection. If the pace remains the same, we could see 239 weeks go by before the collection phase is complete.

In other words, we are looking at an additional 3 to 4.5 years for the data collection phase of this study to be completed. That’s not data analyzed and results published; that’s just data collection. Unless NIH increases its pace, data collection won’t be completed until May 2021 at the earliest.

But will this study produce a case definition? Dr. Nath addressed this back in March 2016 when he said, “Whether we are able to establish [a case definition] beyond an element of doubt or not, it won’t be from this study.” (emphasis added)

Dr. Walter Koroshetz then commented:

the biologic basis of the illness in most of the illnesses is eventually what moves the definitions and allows break-up in the heterogeneity of a disorder.

It’s a particular biological entity. That process is a long process. And so, I would urge people to basically stay the course there. Clearly this protocol which is looking currently at 40 patients – what they find will then need to go into another stage to be validated in other groups of patients to make sure that it’s a real finding that can be generalized. (emphasis added)

In other words, the Clinical Center study will not produce a new case definition. The results may get us closer, but as Dr. Koroshetz points out, it will be a long process.

NIH won’t put a firm timeline on all of this, of course. But I will make a firm prediction: If NIH is going to wait for better diagnostics, better patient characterization, and for Nath’s findings to be validated in a larger group, then NIH won’t return to the case definition issue for at least a decade.

To summarize: NIH is using the three Collaborative Research Centers (but only three) as seeds to generate interest, and will wait for more research applications to come in as a result. NIH’s slow approach to case definition is going to take ten years (or more) to show results.

But we are NOT powerless!!!!! Not by a long shot. In my next post I will share some ideas on what we can do.

 

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One Dance: Millions Missing 2018

I was never a talented dancer, but I loved it.

I quit ballet lessons when I was in the 3rd grade because it was hard. I struggled to learn the simple choreography in school musical productions. I never learned to swing dance or czardas. When I did dance, I looked like that little girl in the Charlie Brown Christmas special. Even in my wildest dreams, I couldn’t dance to Beyonce like these women.

I made up for this lack of talent with enthusiasm. I didn’t clean my apartment; I dance-cleaned. I didn’t move to the beat; I shook my booty (when no one was watching).

Then I got sick with ME, and my last dance was at my wedding. I had been sick for more than a year by then, but the doctors were still telling me I would get better. I was not about to let this disease take my wedding away from me. So I danced with my husband (At Last) and my Dad (Sweet Baby James) and my Grandpa. I even danced with my friends, a little.

My husband and I celebrated our 22nd anniversary a little over a week ago. We haven’t once grooved to a beat or even slow danced in the kitchen in all that time. I couldn’t dance at my brother’s wedding, either. I couldn’t go to my Dad’s wedding reception, let alone dance at it.

Twenty-two years together, without a single dance.

My illness might have been preventable, but not by me. I could not have prevented it by using a condom or by avoiding under-cooked food. Wearing a seat belt would not have prevented my disability. Nor was it the inevitable result of a genetic defect or the typical aging process.

My disability was made possible by the failure of the scientific and medical systems to address this public health crisis. There has been grossly inadequate investment in research, and there is also a history of disinformation and stereotyping perpetuated in research and healthcare. This was true in the 1980s. It was true in the 1990s, when I got sick. And in the 2000s. And it is still true as we approach the end of the second decade of this century.

If you see ME, you are watching a disaster advancing before your eyes. It’s not a disaster because the powers that be are simply unaware of it; they know. And it’s not a disaster because ME is a difficult disease to unravel. After all, cancer is a difficult disease to unravel. What can we do about complicated problems? We invest the resources needed to solve them.

ME is an unsolved mystery because the biomedical research enterprise has consistently refused to invest the funding and expertise needed to figure it out.

NIH points out that it has nearly doubled its investment in ME research from 2016 to 2017. But even NIH has admitted that ME funding must be 10 to 20 times its current level. Compared to the need, NIH funding went from .04% 4% of the need in 2016 to .07% 7% of the need in 2017.* In other words, double of practically nothing is still practically nothing.

While NIH is waiting for a round of grateful applause from people with ME, it is the people with ME who suffer. We disappear from our lives; we struggle to get through each day; we lose careers and dreams and families. We die.

We miss our old lives. We miss big things like career milestones and family events. We miss mundane things, like driving or running errands or walking around the block. We miss the freedom that healthy abled people have to simply do whatever their bodies and minds want to do, without making themselves sick.

We miss being able to dance with our loves. Even just once in twenty-two years.

Today is a global day of protest by and for the Millions Missing. I am missing something that is both small and as unattainable as the sun.

Just one dance.

 

*Edited May 18, 2018 to correct mathematical errors.

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All The Time and None At All

Dr. Francis Collins, in answering a question about ME research on April 20th, said that NIH recognizes and empathizes with our suffering and our “frustration that so little is known.” Then he dropped some knowledge.

Dr. Collins said, “Research, done correctly, takes time.” Thank you, Captain Obvious.

We KNOW research takes time, Dr. Collins. That is why we have been pleading with NIH to invest appropriately in ME research for more than thirty years.

We have felt every second of those years go by, as the sands dropped grain by grain through the hourglass like so many drops of blood. I have personally watched the last 8,603 days (and still counting) of my life dissolve behind me, along with the dreams and plans of the healthy twenty-six year old woman I was before ME derailed my life.

In fact, there has already been plenty of time. Time for me to contemplate the wreckage of my life and try to build something new. And plenty of time for that research – being done correctly – to find some answers. Can you imagine, Dr. Collins, if there had been more research over the last 8,603 days? Don’t you think that more would be known?

Of course, research – done correctly – takes more than time. It takes researchers and research subjects. It takes money. But you know that, don’t you? Because when the President asked you in March 2017 to direct the resources of NIH towards the opioid abuse epidemic, you didn’t tell him that you empathized with his frustration and that it would take time. No, you formed thirty new partnerships in 10 months to find new addiction treatments and alternatives to opioids.

And last month, when Congress gave NIH an extra $3 billion over last year’s funding (instead of the 22% cut proposed by that same White House), you didn’t say No thanks, we just need more time. Not at all. Instead you personally thanked the House Appropriations Subcommittee for the “incredible increase,” as you assured them that, “NIH has immediately set to work to invest those additional resources into groundbreaking research.”

I am not suggesting you should ever bite the hand that feeds you, Dr. Collins. You would not have lasted a quarter century at NIH if you did. But I don’t think you ask every person with a disease to simply bide her remaining time on earth while research can be done correctly.

In one interview, you talked about your long-term family friend from Michigan who is participating in an NIH trial. She has survived more than three years with glioblastoma thanks to immunotherapy. You said, “She’s never been herself since the surgery and the radiation, but she’s alive. I don’t know how to balance those things off. Her husband thinks it’s great that she’s alive, and I can’t argue with that.”

I can think of some people who would be grateful if their loved ones with ME were still alive. Did you tell your friend Dr. Ian Lipkin that Vanessa Li should have drawn comfort from the fact that research was being done correctly, even if it was taking time? Would you say that to the family of my friend Anne Ortegren, a woman who understood that science requires more than time? I can also think of some people with ME who might run out of time. Have you told your friend Dr. Ron Davis that you empathize with his family’s suffering and frustration, but after all, research must be done correctly and that takes time?

So how about we cut the condescending crap. We all know research takes time, as well as money and researchers and innovative thinking. For most of the last thirty years, NIH has only offered us time to watch the grass grow around the ME sinkhole. NIH has had plenty of time, while I have had none at all.

Don’t pat my hand and tell me you care. Don’t tell me that you empathize with my suffering while NIH does a tiny bit of research – correctly, of course.

Stop wasting my time. Do your job.

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Return on Investment: David Tuller

When journalist David Tuller asked for crowdfunding in 2017 to support his research and writing on ME for a year, he had already demonstrated his commitment to a rigorous examination of the many problems and issues associated with the PACE trial. That crowdfunding campaign was successful in part because of his track record and determination to continue working on this story.

Tuller has now launched a 2018 campaign to fund his work for another year, and I believe he has more than proven that he is worth the investment.

At the beginning of 2018, Tuller published a progress report of his work from the first half of the one-year commitment he made. In addition to posts on Virology Blog, Tuller spoke on panels, co-authored a piece with Julie Rehmeyer, wrote letters to multiple scientific publications and traveled to conduct extensive interviews. He also withstood a complaint by Dr. Esther Crawley’s institution about his journalism. Media and public pressure generated by Tuller, Unrest, and the scientific community led to changes in CDC’s recommendations on CBT and GET and the planned review of NICE treatment guidelines.

Since that update in January, Tuller has reported even more developments. The Netherlands may drop the GET treatment recommendation. A brief debate on the PACE trial was held in the UK Parliament. And Tuller has co-authored several articles for peer reviewed publications.

Tuller does not take sole credit for any of this. But there is no doubt in my mind that his work does deserve a solid share of the credit. Investigative journalism is a powerful tool, and Tuller is a highly skilled journalist.

There is something else you should know about Tuller: this is personal for him. He may have started this journey as a neutral observer, but the PACE-Gate scandal shocked him into action. Tuller has already committed to continue working on this story for another year, regardless of whether his current fundraiser fully succeeds.

No one should take this commitment lightly. For a journalist of Tuller’s experience and caliber to devote half of his time for a year is rare in the world of ME. And Tuller has already demonstrated that he offers a great return on our investment.

Tuller’s crowdfunding goal is $75,000 by April 30th and he is already almost halfway there. I hope you will join me in making a contribution, if you are able to do so. And regardless of your financial support, please read and share David Tuller’s work. He can help us keep the pressure on and put the bad science of PACE behind us.

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