It’s rare that ME/CFS as a disease category experiences a “first” that’s positive. Here’s one worth celebrating: the March 11, 2013 announcement by the Food & Drug Administration (FDA) that ME/CFS would be the first condition explored through the agency’s . In most of the regulatory framework that guides FDA decisions, information about diseases of all types comes from industry representatives and clinical researchers who are far-removed from the real-world experience of living day-to-day with a condition. FDA’s new initiative is designed to collect information and perspectives directly from patients, their caregivers and advocates. It was mandated by a broader law passed last summer; its implementation has been a , involving dozens of groups and individuals across a wide spectrum of conditions, including ME/CFS through my participation as one of the process consultants.
More than 90 disease areas that fit a set of specific criteria were submitted for consideration, “competing” for 20 focused workshops to be held quarterly over the next five years. FDA reported receiving 4,500 comments from the public after sharing a draft list of 39 conditions being considered, of which CFS was one. An at FDA provided a forum for public discussion about the criteria and proposed conditions. CFIDS Association Board chairman Amy Squires gave a statement supporting ME/CFS as a final selection, as did four other ME/CFS advocates. We submitted a formal statement to the written record. The March 11 announcement that ME/CFS had been named as the first condition was followed on April 12 by a list of selected for the first three years of the program. Fibromyalgia and irritable bowel syndrome, conditions that frequently co-occur with ME/CFS, also made the list.
Because this Patient-Focused program is new to FDA, there is some uncertainty about how the information FDA collected in written form (through an online portal called a “docket”) or at the April 25-26 workshop will be used by FDA to assess progress at the various stages of drug development. But the effort leading up to the workshop and the dialogue that unfolded there offer good reasons to be optimistic about deepening understanding of what it’s like to live with ME/CFS and how serious the unmet need is for medical treatments. Below is a summary of the workshop; the full recording is available (with subtitles) on the FDA’s website at http://bit.ly/15gdsEV. The recording is broken into segments, making it relatively easy to select specific presentations; here”s the .
DAY ONE: Refreshing Participatory Format
The two-day workshop began on the afternoon of April 25 with introductory comments from three FDA representatives, Dr. Sandra Kweder, deputy director of the Office of New Drugs; Dr. Theresa Mullin, director of the office of planning and informatics and Dr. Sara Eggers, a member of Dr. Mullin’s staff. Dr. Eggers facilitated the session which was dedicated to gaining insight into two broad areas of patient experience:
- Disease symptoms and daily impacts that matter most to patients
- Patients’ perspectives on current approaches to treating CFS and ME
A five-person panel kicked off a broader discussion that invited individuals living with ME/CFS, caregivers and advocates to address the first question. Panelists were drawn from volunteers who submitted their interest in participating through the online registration process. I was a member of the panel and presented top-line results of the 1,300 responses to our patient-focused survey question about life impact. Fellow panelists Dr. Jon Kaiser, Joe Landson, Denise Lopez-Majano and Charlotte Von Salis offered their personal perspectives on life impact. After each of the panelists spoke, Dr. Eggers invited members of the audience to share their experiences. She posed various questions and asked for a show of hands to gauge agreement with symptoms and impacts first raised by panelists. She also probed for more clarity about specific symptoms, like whether a “crash” provoked by physical activity felt the same as one brought on by mental activity. Various dimensions of cognitive impairment – such as word-finding difficulty, trouble with decision-making, inability to multi-task – were illuminated. The conversation was rich and participation was broad and varied.
A second panel comprised of Mary Dimmock, Tasha Keleman, Matina Nicholson, Dr. Mary Schweitzer and Amanda Simpson. They offered short commentary on approaches to treatment, again providing a foundation for the facilitated discussion with audience members led by Dr. Eggers. In addition to various drug therapies used to manage symptoms, the discussion highlighted pacing, alternative and complementary approaches, use of adaptive devices and “rescue” strategies like IV saline. Dr. Eggers posed questions like, “How long do you try a treatment before deciding if it works or not?” and “Which symptom or symptoms are the toughest to manage with current therapies?” Again the diverse perspectives offered enriched understanding of these vexing issues for people living with a condition for which there is no approved treatment and no widely used standard of care.
An open comment period gave an additional 19 people the opportunity to offer brief remarks about any of the topics raised in the earlier period. Among those giving testimony were four well-known expert physicians who spoke to the need for better treatment options for their patients: Drs. Derek Enlander, Dan Peterson, James Baraniuk and Charles Lapp. Dr. Mullin made closing remarks, expressing her thanks to all the attendees for their candor and willingness to share their experiences. A reception hosted by Speak Up for M.E. gave attendees a chance to extend conversations and renew friendships.
DAY TWO: Exploring Drug Development
The second day was dedicated to drug development and regulatory issues. Dr. Theresa Michele, clinical team leader for the division that reviews drug applications for CFS and ME at FDA. Dr. Michele also represents the FDA on the federal CFS Advisory Committee. She began the day by complimenting the “amazing and articulate” contributions made the previous day, expressing how much she had learned from the discussion about what it’s like to live with the condition. Speakers and panelists for this day were selected by FDA with input from various stakeholder groups, including the CFIDS Association.
The day’s presentations were broken into four sessions, each of which concluded with a moderated question and answer session. The first panel was dedicated to innovation, expedited pathways and regulatory considerations. Bernard Munos (InnoThink Center for Research in Biomedical Innovation), Dr. Suzanne Vernon (The CFIDS Association of America) and Melissa Robb (FDA) addressed these issues in 20-minute presentations. In particular, Dr. Vernon reported on the Association’s drug repurposing partnership with Biovista to explore novel uses for medications approved by FDA for other conditions.
The second panel included clinician and patient viewpoints about symptoms and treatments. This panel was asked to respond to issues raised the previous day and to amplify and/or clarify the varied testimony that had been offered. This panel included Dr. Lucinda Bateman (private practice), Dr. Lisa Corbin (University of Colorado), Dr. Lily Chu (patient), Dr. Jose Montoya (Stanford University), Jennifer Spotila (patient), Christine Williams (patient).
Following a lunch break, another set of presentations was given on the topic of clinical trial design and endpoints. Dr. Peter Rowe (Johns Hopkins University School of Medicine), Dr. Christopher Snell (Workwell Foundation), Dr. Elizabeth Unger (CDC) and Ashley Slagle (ORISE) addressed these topics, offering experience from past clinical trials, studies under way and regulatory practices.
The final session was a roundtable discussion involving a large panel, the members of which were asked to summarize issues and help chart the path forward to more and better treatment options. This panel was composed of Dr. Chu (patient), Dr. Jordan Dimitrakoff (Tufts University), Dr. Nancy Klimas (NOVA Southeastern University), Dr. Nancy Lee (Department of Health and Human Services), Dr. Susan Maier (National Institutes of Health), Dr. Michele (FDA), Robert Miller (patient) and Jody Roth (Eli Lilly). One of the important next steps identified during the discussion was the need for a – a regulatory “roadmap” – for industry interested in developing drugs for CFS and ME. Dr. Michele stated that FDA had already begun thinking about developing such guidance and that the information collected during the workshop and in other venues provided a strong platform to work from. She set the expectation that creating such a roadmap would likely require gathering additional input and evidence and might take several months. Ms. Roth echoed the boost to industry’s interest that would be likely to follow FDA’s direction.
Dr. Kweder closed the session and the day by thanking the speakers and participants – both in the room and watching via webcast – for their fortitude and excellent contributions, remarking that the meeting had “met and exceeded” FDA’s expectations. She noted the passion for making a difference in the lives of patients that was evident over the two days. She emphasized the need for collaboration and personal leadership to make progress together.
The workshop was covered by three specialty media outlets that serve the pharmaceutical industry, , and BioWorld. The full-text of these in-depth articles sits behind costly paywalls but these publications have high visibility within the industry and among biotech investors and policymakers. The journal Nature Biotechnology also included a brief report in its May issue.
A comment expressed frequently over the two days by various agency representatives was how much they learned about ME/CFS through the different approaches used to elicit personal experiences, clinical perspectives and lessons from past studies and innovative models.
FDA’s March announcement prompted our swift action to help educate the community about the drug development process and prepare for the meeting that would be a test-case for 19 others. On March 18 we announced a series of six webinars and a patient-focused survey based on the questions FDA had posed and others we believed are important to better understanding the patient experience. To date we’ve collected valuable information from more than 1,400 individuals interested in ME/CFS, nearly 90 percent of whom have been diagnosed with the condition by a health care professional. We”ll post an analysis of interim survey results here soon; the survey remains open so please complete it now and/or share the link. Five of the six webinar recordings are archived on our SolveCFS YouTube channel and have been promoted by FDA and other organizations now preparing to participate in this patient-focused drug development initiative.
We are continuing our outreach and the dialogue with FDA and other collaborators dedicated to expanding the treatment arsenal for ME/CFS. Our Research Institute Without Walls is the world’s first virtual institute dedicated to finding safe and effective treatment for ME/CFS and we’re fully committed to advancing a patient-centered approach to discovery and development. We invite you to stay engaged and active through the SolveCFS BioBank, making a gift to our research program and keeping up to date on the latest news through our Research1st e-newsletter, Facebook, Twitter and YouTube. You are at the center of all we do.
Kim McCleary is CEO of the CFIDS Association of America.