Last week, the CFIDS Association announced the formation of a Research Advisory Council and its newly appointed members. A close look at the roster reveals some interesting signs about the direction the Association may be headed, and it may surprise you.
What is the Research Advisory Council?
In 2011, the Association created a Scientific Advisory Board (pdf) with members from inside and outside the ME/CFS community. The SAB provided informal guidance to the Association’s Board of Directors and staff on issues related to research strategy. It appears that the new Research Advisory Council (RAC) will fill the same role, but the new name reflects the fact that more than scientific expertise is needed. Dr. Suzanne Vernon told me, “We invited people with expertise in regulatory, intellectual property, legal and privacy issues. Since this required people in addition to scientists, we changed the designation from Scientific Advisory Board to Research Advisory Council (RAC).”
The RAC will “advise on research matters as it concerns our strategic plan and the roadmap for our 3-year strategy.” Like its predecessor, the RAC is a volunteer advisory group, and it will not evaluate or approve grant proposals. Dr. Vernon said that the RAC will meet 2 to 4 times a year, in addition to individual consultations, and that the first in-person meeting between the RAC, staff and Board of Directors is planned for March 2014 at Banbury/Cold Spring Harbor.
Some Stay, Some Go
Six members are departing from the SAB, including prominent clinicians and scientists. Dr. Cindy Bateman and Dr. Nancy Klimas are leading ME/CFS clinicians. Dr. Lin Chang (IBS and gastrointenstinal expert), Dr. Robert Silverman (virologist), and Dr. Vincent Racaniello (virologist) are all prominent in their fields, and brought much needed outside expertise to SAB discussions. Dr. Dimitris Papanicalaou is an endocrinologist who did ME/CFS research before going to Merck Research Laboratories. Their departure means the loss of some important expertise, so I asked Dr. Vernon why these members were departing. Regarding Drs. Bateman and Klimas, she told me, “We will be partnering to conduct clinical trials and we need to be able to work with expert and respected ME/CFS clinicians as our clinical collaborators. We did not want there to be any possibility of conflict of interest.” While Dr. Silverman and Dr. Racaniello are leading virologists, they “felt they did not have the clinical and regulatory expertise” to advise the Association on the design and execution of clinical trials and a regulatory strategy.
Six members of the SAB will continue to serve on the RAC: Dr. Italo Biaggioni (expert on the autonomic nervous system); Dr. Gordon Broderick (gene expression and computational models); Russell Bromley (nonprofit strategy consultant); Paul DeStefano (biotech intellectual property attorney); Dr. Roger Dodd (Vice President of Research at the Red Cross); and Kelly Moren (strategic planning consultant).
New Members
Six new members of the RAC bring an interesting mix of expertise and perspectives. Several of them are familiar to the ME/CFS community. Dr. Peter Rowe is one of the most highly regarded ME/CFS clinicians, and the leading expert in pediatric ME/CFS. Kim McCleary is well-known to the community, and her appointment to the RAC was announced at the same time as the announcement of her departure as CEO of the Association. Dr. Terry Tyler is the parent of an ME/CFS patient and a current member of the Association’s Board of Directors. These three members represent a cross-section of the ME/CFS world: a clinician, an advocate, and a family member.
The other three new members come from the pharma world, and this is where things get really interesting. Dr. Vernon told me, “Our research strategy is to bridge the gap between basic and clinical research. . . . These 3 RAC members have knowledge and expertise in translational research and what type of regulatory framework is necessary in order to make that translational as effective as possible.”
The first of theses three new members is Bernard Munos, who may be familiar to some readers. Munos worked inside the pharma industry for years before leaving to reform it from the outside. He has been one of the pioneers in rethinking how drugs should be developed and is a Fellow at Faster Cures. You may remember that he spoke at the FDA’s ME/CFS Drug Development Workshop in April (see the video here). I found Munos’s presentation to be eye-opening, and one of his comments sums it up:
And the traditional drug R&D model is not really suited to tackle something like CFS. . . . Scientists tend to follow the path of least resistance. I mean it’s smart of them. And so if you have the infrastructure, the tools, the data, that makes it easier and cheaper and faster for them to work on your disease, that’s where they will flock. They follow data for reason that we will talk in a few minutes. So we need data. We already have some, but it’s not enough and it’s not in the right format and it’s not good enough, so we need more and better data. We need tools to go along with that data. We need partners. We need money. And lastly, we need leadership and passion. And I should stress already that we have quite a bit of that already, but we need to maybe connect it a little bit better than it is connected today.
Given Munos’s prominence in the field and his cutting edge thinking, it is quite a coup for the Association to recruit him as a member of the RAC. In my opinion, this is a strong signal that the Association is thinking creatively about ways to bring treatments to patients faster.
Another exciting creative signal is the inclusion of Dr. Tomasz Sablinski on the RAC. Sablinski is another big pharma insider who is trying to push the boundaries from the outside through Transparency Life Sciences. The TLS drug development model is based on crowdsourcing. Researchers, clinicians and patients all provide input on the design of clinical trial protocols in an open source format, and can monitor the progress of the trial and see aggregated data. Remote monitoring is used during the trial to reduce the amount of time patients spend in waiting rooms and office visits. TLS is currently working on MS and Parkinson’s (a protocol for low-dose naltrexone in Crohn’s was discontinued because of intellectual property issues). The trend in clinical trials and drug development is to capture patient input and experience earlier in the process, and having patients participate in actual trial design is revolutionary. I think this is a wonderful perspective to bring to ME/CFS.
The final new RAC member, Dr. Mark Demitrack of Neuronetics, is a more controversial choice. Demitrack is a psychiatrist and expert in clinical trial design, having led the post-marketing development of Effexor. He also helped shepherd transcranial magnetic stimulation therapy to FDA approval for clinical depression. Earlier in his career, Demitrack conducted research on cortisol levels in CFS and fibromyalgia (abstracts here and here). In 1996, he co-authored Chronic Fatigue Syndrome: An Integrative Approach to Evaluation and Treatment. I have not read this book, but reviews on Amazon and elsewhere are very negative, and state that the book posits that CFS is caused by depression and stress. In 2005, Demitrack participated in the CFS Computational Challenge meeting co-sponsored by CDC and The CFIDS Association, and gave a presentation on “Clinical Perspectives on Therapeutic Interventions for CFS.” Banbury requires that meeting discussions remain confidential, so there is no report from this meeting. However, in the 2006 issue of Pharmacogenomics where the Computational Challenge papers were published, Demitrack published a paper entitled, “Clinical methodology and its implications for the study of therapeutic interventions for chronic fatigue syndrome: a commentary.” There isn’t space to analyze the article fully here, but Demitrack does seem to have stepped back from a purely psychological theory for ME/CFS and adopted a multi-factoral neuroendocrine view of the illness. The article focuses on the importance of clinical trial design, selection of measurements, homogenous cohorts, and understanding prior treatment response. I asked Dr. Vernon about the significance of appointing a psychiatrist to the RAC and the implications for the Association’s view of the disease. She responded, “Dr. Demitrack is expert in study design, clinical research and regulation. He understands that the CAA requires a regulatory framework in order to bring safe and effective treatments to ME/CFS patients, the core of our 3-year strategic plan and research strategy. The CAA does not view ME/CFS as a psychological disorder.” Speaking for myself, I think it will be important to keep an eye on Dr. Demitrack’s views on ME/CFS and the impact he has on the Association’s research strategy.
Roadmap
The membership composition of the RAC is a mix of expertise and and perspectives. There is an ME/CFS clinician, an advocate, a family member, an attorney, two ME/CFS researchers, two strategy consultants, a nonprofit research leader, and three pharma experts. Clearly, the Association has stacked the RAC with people who can provide advice on research and regulatory strategy for bringing treatments to patients. Several of the members are recognized as cutting edge thinkers about treatment development. The perspective of an expert clinician and a family member should keep views of patient experiences on the table as well. It’s also an endorsement of the Association’s strategy for prominent people like Bernard Munos and Dr. Sablinski to get on board, as I expect that they both receive plenty of invitations from companies and nonprofits.
So where is the Association headed? The RAC will provide expert advice on the Association’s research and regulatory strategies. The PCORI grant, and the partnership with Patients Like Me, would fund a data collection network governed by patients. And the partnership with Biovista will soon begin a clinical trial. These are strong signs of what may be in the 3 year strategic plan mentioned by Dr. Vernon, and the signs point to the development and testing of treatments to get them in the hands of patients as soon as possible.
If You Want Our Support
A week ago, ME/CFS patients found out about HHS’s plan to sole source a contract to the Institute of Medicine to create a consensus case definition for our disease when the sole source notice was published. Today, the CFS Advisory Committee listserv finally got around to telling us about it. Here’s what they said:
Several things strike me as curious about this email, apart from the obvious point that it is a week too late. First, the email refers to the 2011 CFSAC recommendation and not the 2012 recommendation that said a workshop of ME/CFS experts should be convened to consider a definition, starting with the Canadian Consensus Criteria. It’s curious to me that they fail to mention it, particularly since it is one of the Committee’s High Priority Recommendations. Second, the email says the IOM will bring together experts, the community and other stakeholders. Curious, again, because several different sources have suggested to me that this plan to engage the IOM was intentionally concealed from the patient community.
If CFSAC – or more specifically, HHS – wants our support for an IOM process to create a clinical case definition, then they should be willing to address our concerns. I sent the following email to CFSAC with some specific requests:
I will report any response that I receive.