The U.S. Food and Drug Administration (FDA) will host a workshop on drug development and CFS and ME on April 25-26, 2013. This FDA workshop will also serve as the first of 20 disease-specific meetings where the FDA will gather patients” perspectives as part of a mandated “Patient-Focused Drug Development Initiative.” Learn more about the FDA workshop in the FDA”s March 11, 2013 Federal Register notice or in this post on Research1st: http://bit.ly/fda-mtg-agenda.
The CFIDS Association of America believes this is a historic opportunity for the ME/CFS* community to establish ME/CFS as an important priority for drug development. We can also set the standard for patient engagement for other conditions that will follow in the FDA series of 20 patient-focused meetings. Let”s lead the way!
To inform, educate and engage with the community so that our individual contributions have amplified impact, we are pleased to announce a spring webinar series and a special patient-focused survey.
We invite you to complete a new survey based on questions posed in the Federal Register notice for the FDA meeting. The survey, designed for people living with ME/CFS and those closest to them, poses open-ended questions to allow respondents to describe the symptoms, daily impacts that matter most, as well as share personal perspectives on treatment. Questions are worded in the same way the FDA has asked them; we”ve supplemented with some some additional questions on the same topics. You can answer with phrases or lists; you do not have to write complete sentences.
All survey responses are anonymous and confidential. We intend to present an analysis of the collective responses at the FDA meeting and at the May 22-23 meeting of the federal CFS Advisory Committee to better inform the federal health agencies about these issues.
Survey Link: https://www.surveymonkey.com/s/pt-focused-survey
Please feel free to share this post and the survey link with family members, friends and people living with ME/CFS.
We make every effort to record our webinar programs and post the recordings on our YouTube channel for those unable to attend the live event and for later reference. Here”s the schedule of spring programs:
Date/Time (live event): March 28 at 1 p.m. EDT
Speaker: Kristin Schneeman, Program Director, FasterCures
Host: Kim McCleary, CEO, The CFIDS Association of America
More Detailed Information: http://bit.ly/2013-webinar-1
Kristin Schneeman outlined the process to translate scientific discoveries into accessible medical solutions. She defined the major components of the research and development (R&D) cycle as a basis for helping community members understand the landscape for drug development and contrast it with the landscape for basic research. Kim McCleary hosted the webinar and provided additional information about how the CFIDS Association of America is fostering development of safe and effective treatments for ME/CFS. The information included in this program is a great platform for understanding the next two webinar topics, described further below.
- “Getting Started” and “Crossing Over the Valley of Death” (FasterCures publications)
- “The FDA: Past, Present & Future” (video from FasterCures’ Celebration of Science)
- (FDA materials)
- (FDA infographic)
Webinar 2: Finding your strongest voice for public testimony
Date/Time (live event): April 4, 2013 at 1 p.m. EDT
Speakers: Leigh Reynolds (Engagement Manager) and Kim McCleary (CEO), The CFIDS Association of America
More Detailed Information: http://bit.ly/2013-webinar-2
CFIDS Association CEO Kim McCleary described the FDA’s Patient-Focused Drug Development Program and how it ties into the April 25-26 Workshop on ME and CFS drug development. Engagement Manager Leigh Reynolds shared ways to make patient testimony as effective as possible and ways to participate in the drug development process leading up to and beyond the FDA meeting. Both speakers are informed by more than two decades of experience working with patient communities, health agencies and policymakers. The FDA has set April 8, 2013 as the deadline for workshop registration (in person or by web cast), requests to participate on the panel, requests to provide public testimony and submission of written testimony. Written comments after the April 25-26 workshop will be accepted until August 2, 2013. We”ll address these opportunities (and others!) during this webinar to help advocates make the most of them.
- (FDA materials)
- “Back to Basics: HIV/AIDS Advocacy As A Model for Change” (FasterCures publication)
- “ME and CFS Webinar: Working Together for Change” (FDA webinar)
- (video from FasterCures’ Celebration of Science)
- “Tell It to the FDA” (blog post by ME/CFS advocate Jennie Spotila, J.D., outlining how to take part in the upcoming FDA workshop)
Webinar 3: What does the phrase “safe and effective treatment” really mean?
Date/Time (live event): April 11, 2013 at 1 p.m. EDT
Speakers: Kim McCleary, CEO, The CFIDS Association of America
Host: Suzanne Vernon, PhD, Scientific Director, The CFIDS Association of America
More Detailed Information: http://bit.ly/2013-webinar-3
The phrase, “safe and effective treatment,” guides the FDA’s regulatory review of every aspect of drug development. Kim McCleary will break down each word in the phrase to help community members understand how drugs are studied and data is reviewed throughout the approval process. Scientific director Suzanne Vernon, Ph.D., will help put these terms into the context of research and development for more and better ME/CFS treatments.
- (FDA materials)
- (FDA materials)
- (FDA materials)
- “Who Pays for Treatments and Cures?” (video from FasterCures’ Celebration of Science)
Webinar 4: How the CFIDS Association is fostering the development of safe and effective therapies for ME/CFS
Date/Time (Live Event): April 17, 2013 at 1 p.m. EDT
Speakers: Suzanne Vernon, Ph.D. (Scientific Director) and Mark Stone (Director of Development), The CFIDS Association of America
More Detailed Information: http://bit.ly/2013-webinar4
Dr. Suzanne Vernon and Mark Stone described the Association’s venture philanthropy model of being a convener, connector and collaborator to move promising discoveries through the drug development process, keeping the patient as our central focus every step of the way.
- “Discovery of Effective Treatment” (CFIDS Association brochure)
- “Top 10 Reasons to Support the CFIDS Association of America” (Research1st blog post)
- 2012 Report to Catalyst Fund Investors (CFIDS Association report)
- “Honest Brokers: How Venture Philanthropy Groups Are Changing Medical Research” (FasterCures publication)
Webinar 5: Assessment of the ME/CFS Microbiome Before and After Exercise
Date/Time (live event): May 2, 2013 at 1 p.m. EDT
Speakers: Suzanne Vernon, Ph.D., Scientific Director of the CFIDS Association of America, and Sanjay Shukla, Ph.D., Research Scientist, Marshfield Clinic Research Foundation
One-Page Summary: http://bit.ly/microbiome-ME-CFS
Suzanne introduced the study of the human microbiome, millions of one-cell life forms that live within our bodies, and the importance of the microbiome in human health. Dr. Shukla described his research project, funded by the CFIDS Association, studying the gastrointestinal microbiome before and after an exercise challenge. He presented some results from the study and his thoughts about where this data leads next. This is the first ME/CFS-specific study of the microbiome, a very “hot” area of research across many disease states.
**Unfortunately we are unable to post the recording of this webinar. Dr. Vernon will be recording a summary of the presentation which should be posted soon. We apologize for the delay.
- “The Role of the Microbiome in ME/CFS“ (One-page summary of May 2 webinar)
- “Return on Your Investment: Sanjay Shukla, PhD” (Research1st post)
- “Human Microbiota” (The journal Nature”s special supplement dedicated to the microbiome)
- Explore the Human Microbiome (Interactive infographic from Rational Discovery Blog)
- The Human Microbiome Project (NIH resource for its Common Fund projects)
- “Go With Your Gut: The Microbiome is Hot” (Research1st post)
Webinar 6: Epigenetic Changes and CFS: Identifying the culprit
Date/Time (Live Event): May 16, 2013 at 1 p.m. EDT
Speaker: Patrick McGowan, Ph.D., Assistant Professor, Department of Biological Sciences, University of Toronto
Dr. McGowan introduced another “hot” field – the study of epigenetics and why it”s important to understanding human health and disease. He described the influences of environment on the epigenome and why the literature on HPA axis changes in ME/CFS prompted him to want to study the epigenetics of ME/CFS patients. He described his research on glucocorticoid signaling and the preliminary results he’s obtained using samples collected through the SolveCFS BioBank. This is the very first study of epigenetics in ME/CFS; it”s being conducted under our Research Institute Without Walls (RIWW) thanks to the generosity of our donors and BioBank participants.
- “Genetic Changes and CFS: Identifying the Culprit” (Research1st post)
- “Close-Up on the Research Institute Without Walls” (Research1st post)
- “Why DNA Isn’t Your Destiny” (TIME magazine article)
- “Epigenetics – A Primer” (The Scientist article)
*We use the term ME/CFS to refer to conditions also known as chronic fatigue syndrome, CFS, myalgic encephalomyelitis, myalgic encephalopathy, ME and by other names.