Chairman: For open session, this is a concept clearance, and just to remind you, normally we’re expected to spend our appropriated funds, sort of open to all things within our mission, and if we decided to a priori say we’re going to set aside a pot of money for some particular topic, then we need to get some kind of concept clearance for doing that.
And so that means that we’re going to describe to you the basic purpose, scope and objectives of what a potential solicitation might be. So this is just a concept: you won’t be getting details about exactly how things will be solicited but the concept of what the particular goals are.
And so once we have that clearance then it is not uncommon that cleared concepts end up turning into RFAs or RFPs or other specific solicitations. What we want from Council is some feeling that you think that this is a sufficiently important goal that it would be worth setting aside some money to solicit for applications to accomplish those purposes.
So with that, let me then introduce Vicky Whittemore, who is Program Director in our Channels, Synapses and Circuits Cluster, and she is also the leader of a Trans-NIH Working Group on ME/CFS. And it’s been an interesting activity for her.
So Vicky, I’ll turn it over to you.
Vicky Whittemore: Right, thank you. So what I’m going to do today is to present a concept to you for research on myalgic encephalomyelitis/chronic fatigue syndrome, or ME/CFS, and ME/CFS is a very debilitating disease, with many of the individuals becoming ill post-infection and then becoming chronically ill over very long periods of time. I’ve met patients or individuals with ME/CFS who have been sick for more than 30 years.
There is no known etiology, there are no FDA-approved treatments, and it’s really a research area that is in desperate need of bolstering the research infrastructure and training.
So we are presenting this concept with several goals: to support research and to develop research capacity; to stimulate research, especially on the etiology of the disease and biomarkers of disease, as well as to develop a large, well-defined cohort that could then be utilized in future clinical trials, as well as to train young investigators.
And one of the things that was very startling to us when we did a portfolio analysis was to realize that not only is NIH – and this means any institute at NIH – not funding any training grants, so no Fs, no Ks, no Ts, we’ve never even received an application in those categories, which is pretty shocking.
So there’s significant need for building research infrastructure, for bringing in the ability to train, and to do research that involves multiple sites. And so our concept is that we would put in place a consortium with multiple sites utilizing common protocols across the clinical projects who can also then develop studies across the sites that would address etiology, potentially imaging, potentially biomarkers studies, genetics, that would all work together.
Because one of the other issues that we’ve seen in this research area is that the research that’s been funded and done by investigators typically involves fairly small cohorts of individuals with the disease and that there’s no generalization across and no way to get… group these patients.
So we would fund a consortium initially with several sites, together with a data-managing coordinating center that could help with… to standardize protocols, manage the whole consortium, with the ability then to either add additional consortia and/or additional sites to the consortia in future years, that would help to build this infrastructure that we could then also, down the line, implement clinical trials across the consortia utilizing these academic centers.
One of the things that’s also very interesting about this area is that the majority of clinicians and healthcare providers are not located at academic institutions. They are located in private practices, and so there really has not been that partnership between the clinicians and research investigators.
So one of the things that we worked very closely with our friends and colleagues at the National Center for Accelerating Translational Science, NCATS, is to think about how each of these sites could also then partner with the CTSAs [Clinical and Translational Science Awards] that could provide infrastructure and support and the ability to train as well as some of the other initiatives that their institute… that can help to support patient recruitment and clinical-trial readiness across this consortium.
So I have to acknowledge the individuals from the 24 different institutes and centers who make up the Trans-NIH ME/CFS Working Group, and especially Joe Breen who’s here from NIAID, Cheryl Kitt who’s here today from OER and again my colleagues in NCATS Todd Wilson and David Eckstein who’ve been especially helpful in helping us think this through.
If this concept is approved today, what we would do is move forward, working with all of the institutes to look at specific areas of interest that fall within the mission of each of the participating institutes, and joint funding of this consortium and the data management center. So I’ll stop there and see if anyone has any questions or comments.
Dr. Janet Hieshetter: What you describe sounds very similar to the NCATS Rare Diseases Clinical Research Network…
Hieshetter: …which involves patient advocacy groups…
Hieshetter: …and I think they had a really important role as it relates to patient recruitment and education and also just general support, so I would encourage you to think about a role for PAGs [Patient Advisory Groups] in the model that you’re putting together now.
Whittemore: Yes, I failed to mention that we already have considered that, that patient engagement would be a large part of the consortium as well. Thank you for that comment.
Other questions or comments?
Dr. Amy Brooks-Kayal: I just wondered, Vicky, is a part of this… are there very well defined criteria at this point for this condition? I mean, I think that’s been one of the areas that’s really held back research in this area, is that I think there are a lot of varied and often incorrect assumptions about what this disorder is.
Whittemore: That’s a very good question, and no, there are not. So as you may know, there are many different diagnostic criteria that exist out there that are more or less broad and capture more or less broad groups of patients.
One of the projects that we’re launching this summer, actually, is a project together with the CDC and FDA to look at common data elements that would be able to measure common elements across these patients regardless of the criteria that’s being applied so that we can begin to subtype and really look at some of these individuals, and groups of individuals, that fall into this what I think is probably a large spectrum of diseases but have been lumped into this category of ME/CFS.
#MEAction would like to thank the patient-volunteers who transcribed this material.
In August, we shared with you that we and six other ME/CFS organizations had submitted a proposal to the National Center of Health Statistics (NCHS) to fix the coding of ME/CFS in the US International Classification of Diseases (ICD-10-CM). Today, we are writing with an update on that proposal and asking that you sign the