The feds have been stating that ME/CFS is a very serious disease – that it’s a priority – that they’re REALLY committed to helping people with ME/CFS, for years. I’ve never heard anyone at the NIH ever state they believe ME/CFS is a minor disorder.

proof is in the pudding

A nice, rich “Capitol Hill” pudding. Will the feds pudding for ME/CFS be as sweet?

The proof, though, is always in the pudding. The takeaway message from years of observing “fed-speak” is that only action counts. There’s certainly reason to hope.

A lot of promises have been made. The director the NIH himself, Francis Collins, has been very supportive.

It’s time, though, to check out the pudding….

We got our first glimpse of the NIH and the Department of Health and Human Services new orientation to ME/CFS in their response to the CFSAC’s recommendations. CFSAC, the federal advisory committee for ME/CFS, provides regular recommendations to the NIH and other agencies. They clearly put a lot of work into the latest recommendations. The NIH and other agencies often make some movement on some small recommendations, but rarely do anything about the bigger ones.

This time, there was reason to expect things would be different. The IOM and P2P reports were released, the NIH has money to spend, and Francis Collins, the picture of sincerity, has pledged that the NIH would move aggressively to increase funding for ME/CFS. Just watch us, he said.

We are.

CFSAC Recommendations

#1 Patient Registry

CFSAC recommended that the NIH create a patient registry where ME/CFS grant recipients would park their data. Importantly, the investigators would be required to use common data elements. That would fulfill the AHRQ and P2P reports goal of standardizing studies so that researchers wouldn’t be comparing apples to oranges. ME/CFS has enough problems with its heterogeneous populations without researchers gathering data that no one knows how to make sense of.

patient registry me/cfs

The feds agreed that a patient registry is important but refused to support building one.

DHHS Response: The DHHS agreed that developing a patient registry is very important for the field, but it’s not apparently going to be done by them. Citing the “limited funds” available at the NIH for developing non-hypothesis driven projects the DHHS turned CFSAC down, and suggested ME/CFS start looking to patient support organizations instead.

If NIH’s goal is to fulfill the needs the P2P and IOM reports laid out, then a Patient Registry that requires standardized inputs would be a great beginning.  Remember that we’re talking about addressing decades of neglect in a field that the recent federal reports made clear needs to be rebuilt to succeed. It was not to be, though.

It’s not as if the NIH doesn’t fund patient registries. A quick scan found at least a dozen the NIH funds, or has helped build in the past.

NIH Funded Patient Registries

  • National Alopecia Areata Registry
  • Juvenile Idiopathic Arthritis
  • Breast Cancer Surveillance
  • Colon Cancer
  • Osteopathic Sarcoma
  • Breast and Colon Cancer
  • Down’s Syndrome
  • Hematologic Tumors
  • Lupus Family Registry and Repository

The irony is that it’s probably not necessary to build a new patient registry at all.  The NIH pats itself on the back with how many private/public partnerships it uses to maximize taxpayer dollars, and the Solve ME/CFS Initiative has a nice registry they could probably work with. That is apparently not an option here.

  • Grade – F: the DHHS gets an F for not even trying

Request For Applications

CFSAC recommends that targeted Requests for Applications (RFAs), which clearly prioritize the identification and validation of distinct biomarkers and objective diagnostic tests and give preference to collaborative network initiatives, be issued as soon as possible….. CFSAC believes that time is of the essence regarding this urgent recommendation.

CFSAC’s  recommendation for a Request for Applications (RFA) grant is the biggie. A grant opportunity with funds already attached to it, an RFA draws researchers like flies to honey. One worked for ME/CFS in 2006 and it would work ten times better now.

In fact, given the low rates of grant applications for ME/CFS, it’s hard to see how the NIH can do anything significant quickly without an RFA. Remember Collins’ promise to move “aggressively”.  An RFA would fulfill that promise.

The NIH, did, however, what it’s done for decades. It agreed that biomarkers, etc. are vitally needed but then stated that investigator- initiated grants would be crucial to ME/CFS’s success; i.e. it would provide no RFA.

RFA chronic fatigue syndrome

An RFA would bring more researchers into the field quickly – yet the NIH refuses to produce one.

The response suggested that NIH believes that business as usual is good enough. Never mind that business as usual – waiting for investigators to somehow drop out of the clouds and pummel the NIH with ME/CFS grant applications – got us into this mess. It will wait for researchers to come to them.

Why the NIH is not willing to take what appears to be a rather simple step is baffling. The ORWH got an RFA for ME/CFS in 2006 when support for ME/CFS was abysmal. Now, ten years, two federally funded reports that urgently called for funding increases, a $2 billion increase in NIH funding, and Francis Collins’ promises  later, the NIH still will not fund an RFA for ME/CFS. Even a little one…

It’s just weird.

The DHHS could have told CFSAC that they were developing programs to expand the number of “investigator-initiated” grant applications for ME/CFS. That would be something. Even that was apparently too much.

  • Grade: F

Using the NIH’s Clinical Center for Clinical trials.

  • CFSAC Request – Use the NIH’s Clinical Center to conduct clinical trials
  • NIH Response – Use the NIH’s Clinical Center for a research study.

The NIH’s clinical center research study is a notable step forward. It’s the one concrete step that’s been made so far.

“The investigators believe that intensive clinical studies of a well-characterized subgroup of individuals with ME/CFS will be essential to gaining insight into the underlying mechanism(s) of ME/CFS. Greater insights into the pathophysiology of the disease are critically needed to inform development of therapies that will benefit individuals with ME/CFS.” The NIH

Unfortunately the NIH then implied that the field is not ready for clinical trials; that more investigators are needed:

For therapeutic trials a cadre of committed, well-trained investigators will be essential to bring therapies to well-designed phase 2 trials with clear Go/NoGo endpoints. It is likely that a larger group of committed investigators will be required to carry out larger phase 3 trials. NIH

me/cfs practitioners

The NIH gave some indication that it will move forward on clinical trials

It was remarkable seeing the NIH use the word “committed”, given how little commitment it will show in this document.

A cadre of committed, well trained investigators certainly are needed  to carry out clinical trials but the NIH might want to check out their ME/CFS address book; we already have one.  Doctors like Dr. Bateman, Dr. Lapp, Dr. Klimas and Dr. Peterson have been involved in clinical trials for decades. They’ve repeatedly stated they are ready now.

The NIH did make a vague commitment

“NIH will work with the scientific and patient communities to enable the development of clinical trials in the future.”

The commitment is vague but it has promise and with Francis Collins talking about Ampligen and Rituximab trials, it’s encouraging.

  • Grade B (+) – good for the NIH for beginning an intensive study on ME/CFS patients. Bad on the NIH for implying that the field was not ready for clinical trials. Good for them for committing to “enable the development of clinical trials in the future.” Given some reports suggesting that the NIH may, indeed, be serious about Rituximab and Ampligen trials, the NIH gets a B (+)

Standardize Assessment Methods and Measures

Research Definition – CFSAC recommended that the NIH require the use of the Canadian Consensus Criteria in research studies. I believe missed the boat on the research definition question. Recommending that the NIH immediately commit funding to produce a new research definition would have been better.

The CCC may be the best definition we have, but it carries enough baggage (it’s a consensus driven definition and could select out a “pysch” subset) that it’s unlikely the feds are going to sign off on it. Plus, unless I’m wrong, the groundwork for a statistically based research definition the feds would sign off on has been mostly laid. CFSAC’s unwillingness to push the DHHS to produce what is arguably the most needed element in ME/CFS research – a new research definition – is baffling to me.

A "possibility" exists the already overburdened CDC will work with NINDS on producing validated assessments

A “possibility” exists the already overburdened CDC will work with NINDS on producing validated assessments

Establish Uniform Assessment Methods – In a recommendation that could have been taken straight out of the P2P report, CFSAC also recommended that a “methodological workgroup comprised of disease experts and stakeholders to establish uniform assessment methods, etc.

Something to that end may be in the works at the DHHS. The DHHS and CDC reported the “possibility” that the CDC will partner with NINDS to co-sponsor the development of common data elements for ME/CFS.

That would be a good start but with the CDC’s ME/CFS program years behind  in publishing studies from it’s first multi-site study one wonders how much more it can take on – or how quickly it can do anything. Unless the CDC provided more funding, it seems likely that this program would cannibalize resources from other projects as well.

The CDC/NINDS collaboration would be progress but it’s a far cry from the P2P report recommendation that the NIH create a center and a group to work on these issues.

Plus, it’s just a possibility at this point. If the DHHS is serious about moving this field forward they should commit to something concrete – like stating they’re going to produce uniform assessment methods and set a target date.

  • Grade C-  To be assessed after we learn more about the CDC/NINDS collaboration

CFSAC – Assign the Disease to an Institute:

“Given the clear involvement of neurological dysfunction in ME/CFS, CFSAC recommends that the disease be assigned to the National Institute of Neurological Disorders and Stroke (NINDS). CFSAC”

DHHS Response – the DHHS reiterated that NINDS has taken the lead in the Trans-NIH Working Groups. The emergence of NINDS has been gratifying but let’s not kid ourselves; it’s not the same as ME/CFS being based in NINDS or having an Institute be responsible for this illness.  Plus we are embedded in a weak structure.

working group ME/CFS

The Working Group is being tasked with doing something it’s not designed to do – build a vital ME/CFS program.

The idea that a reconstituted Trans-NIH Working Group is still at the epicenter of the NIH’s work on ME/CFS is worrisome. The DHHS’s response to CFSAC’s recommendations sounds like it’s coming out of the old Working Group.

Notice that funding isn’t mentioned in the DHHS’s  description below of what Working Group’s do. Except for ME/CFS, groups like these are never used for funding at the NIH. They’re communication and discussion vehicles that help to optimize the work that Institute’s are already doing.

“This group will provide a forum for coordination of ME/CFS research across all NIH institutes, centers and offices, as well as for discussion of scientific opportunities and gaps in the research that can be addressed by working together to further research on ME/CFS.”‘

Plus the Working Group’s floating leadership position provides little accountability. The end result for ME/CFS is that it’s not in an Institute, and no one person or Institution bears responsibility for it. The setup does not inspire confidence.

The fact that this hodge-podge effort is the best the NIH came up with (after the IOM and P2P reports basically gave them an F in supporting ME/CFS) may indicate how poisoned the conversation around ME/CFS is there. It bears pointing out that ME/CFS is not alone in this regard; the NIH is neglecting every disease (migraine, fibromyalgia, IBS, etc.) ME/CFS has traditionally been associated with.

Grade – F – Not only has the NIH embraced a decision-making process (the Working Group) that has been utterly ineffective in the past, it’s produced what appears to me, at least, to be a weak format for it – a floating leadership position. Time, of course, will tell, with this group.

Create an ME/CFS Czar (or Czarina) to Oversee the Effort

CFSAC attempted to insert some accountability. It recommended that the

HHS appoint a senior-level cross-agency leader (“czar”) with the authority, position and fiscal responsibility required to coordinate, develop, implement, and monitor a broad strategic cross-agency response to this disease through open and collaborative engagement of both internal and external stakeholders.

The buck would stop somewhere if a ME/CFS Czar or Czarina (I nominate Suzanne Vernon) was present but the DHHS asserted that that position is not necessary at this time. (It’s going to go with its no-leadership approach). Appointing an ME/CFS Czar, however, is precisely the kind of bold, “aggressive” move that Collin’s statements suggested might be happening.

Grade – F

Provide Research Funding Commensurate with the Burden of Disease

From the Solve ME/CFS Initiative’s response to the DHHS document: “It is the responsibility of the government to invest in solutions for serious medical problems…without obfuscation and kicking the can down the road”

CFSAC:

To facilitate the above goals, CFSAC recommends that the Secretary work with HHS agencies to ensure that total research funding is commensurate with the epidemiologic prevalence and economic burden imposed by this disease. Based on disease prevalence, equitable funding is estimated to be $250,000,000 per year.

CFSAC provides the right keywords here. Burden of disease has become a big deal in the medical research field, and the NIH has been heavily criticized for not taking it into account when it allocates funding. In its recent strategic plan it pledged (rather begrudgingly) to give it more priority.

If it does then diseases like migraine, fibromyalgia and ME/CFS should get more funding and funding for diseases like HIV/AIDS should decline. HIV/AIDS funding is declining but there’s no indication yet that the historically neglected, but prevalent diseases like migraine, FM and ME/CFS are going to benefit.

The CDC’s and DHHS’s response certainly does not provide any assurance that they will. They implied again – for the umpteenth time – that the ME/CFS researchers are to blame. If only ME/CFS researchers would submit better grant applications, the NIH would be happy to fund them. Absent ME/CFS researchers, their position is of utter helplessness. (Please stop victimizing us with your lousy grant applications!) They stated:

“Public health needs are a critical factor in NIH’s funding decisions, but scientific merit, portfolio balance, and budgetary impact are also important considerations. Often, the funding level is driven by the number of high quality applications that are received. NIH encourages the ME/CFS research community to submit grant applications utilizing new and innovative approaches to study the disease.”

This is like déjà vu all over again. The NIH keeps telling the “ME/CFS community” to submit more and better research grants. ME/CFS advocates respond that the number of grant applications have been low for decades. They assert that an RFA is needed to jump start a nascent field that the research community is wary of. An RFA, they say, would indicate to the research community that the NIH is committed to supporting ME/CFS research. If it does that, the researchers will show up.

thumbs down for NIH for ME/CFS

Yet another thumbs down on an RFA for ME/CFS…and the NIH demonstrating its commitment to meaningful change for ME/CFS

The NIH is, still, however, unwilling to produce that commitment.

Note that the main problem may not be poor grant applications, but that not many grant applications are being received. (An FOIA has been submitted to figure that out).

If that’s the case, then given how critical a role the NIH says “public health needs” matter to it, it should be willing to find ways to increase researcher interest.

The FDA does. In recognition of the special problems regarding drug development for rare diseases the FDA lowers its requirements for drug approval. By doing so it’s been able to bring drugs to people with rare diseases.

The NIH, however, is simply sitting on its hands again. The Feds could have said they were working on a strategic plan to address the shortcomings in ME/CFS funding. They could have said that they were working on ways to bring new investigators into the field.

The bottom line from the NIH, at least at this point, continues to be that you generate it not us. That’s the last thing I expected after Dr. Collin’s promises.

Grade – F-

Validate and Refine the Proposed Criteria within Two Years

This cycle of waiting for related events must be broken by expending the funds to develop the criteria. Surely this is a federal responsibility…The Solve ME/CFS Initiative

CFSAC recommends:

that the proposed Diagnostic Criteria be validated, and refined if needed, within two years by disease experts and/or by the methodological workgroup recommended herein for sensitivity and specificity during different stages of disease and different levels of severity.

The DHHS responded by stating that they agreed with the need to update the Diagnostic Criteria, and that “When to begin the process of updating the guidelines will depend on the availability of such evidence.”

They’re right about new evidence being needed to update the guidelines – but are they going to play a role in producing that evidence?

CFSAC made several more recommendations. Check them out and the Solve ME/CFS Initiative’s response to them here.

Conclusion

These responses perpetuate the status quo and fail to recognize the deep, longstanding chasm between appropriate funding…and current funding. Significant progress cannot and will not be made until the federal agencies recognize that their current funding approaches leave this disease egregiously underfunded. The Solve ME/CFS response

There are some good things in the DHHS’s response. The possibility of clinical trials and the CDC’s possible work with NINDS to produce standardized assessment tool stands out. The research study at the Clinical Center is a big step forward. It is encouraging as well that NINDS has gotten so involved. We have moved forward.

The DHHS response, however, is disappointing. The NIH’s continuing reluctance to produce an RFA – the one thing we know will spark interest in ME/CFS –  is baffling.

Thus far Dr. Collin's words are ringing pretty hollow

Thus far Dr. Collin’s words are ringing pretty hollow

Those IOM and P2P reports were designed to give the NIH and CDC guidance on how to move the field forward. The NIH and CDC asked for them and now it’s incumbent on them to take the recommendations seriously and move forward on them.

This is surely not the response the authors of the reports expected or wanted.

The First Test – Not the Last Test

Of course, it’s still very early in the process. The feds often take a long time to produce something. The new Working Group has met perhaps once or twice.

This was just the first test. The real test will come when they provide a strategic plan to meet the needs of the ME/CFS community. That will tell us everything.

They may surprise us yet.

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