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.”
The Solve ME/CFS Initiative believes this is a historic opportunity for the ME/CFS* community to establish ME/CFS as an important priority for drug development. We’ve launched a special patient-focused survey and six-part spring webinar series to inform, educate and engage with the community so that our individual contributions have amplified impact. Learn more here: http://bit.ly/spring-series
We’ve collected other resources about the workshop here for convenient reference.
The workshop will be held on April 25-26, 2013 at the Bethesda Marriott, 5151 Pooks Hill Road, Bethesda, Maryland.
- Announcement of the workshop in the March 11, 2013 Federal Register: http://1.usa.gov/Y4ytgz
- Draft agenda posted to the FDA website (and copied below): http://1.usa.gov/WVQTC4
- FDA’s page for resources on Drug Development for ME and CFS: http://www.fda.gov/Drugs/NewsEvents/ucm319188.htm
- NEW: Frequently Asked Questions About the April 25-26 Meeting on CFS and ME (posted by FDA on March 25): http://www.fda.gov/downloads/Drugs/NewsEvents/UCM345237.pdf
- April 25 — 1:00 p.m. (EDT) until 5:00 p.m.
- April 26 — 8:30 a.m. (EDT) until 5:00 p.m.
The workshop is open to the public on a space available basis. It will also be webcast live. Advance registration is required to attend either in person or by webcast. There is no fee for registration.
The workshop format is designed to include opportunities for people living with ME/CFS and those close to them the opportunity to share their input and perspectives. The deadline to register to participate on one of the panels or give oral testimony is April 8, 2013.
Written testimony to be considered at the meeting must be submitted by 5:00 p.m. (EDT) on April 8, 2013. The FDA docket will remain open until August 2, 2013 for anyone who would like to submit written testimony before that date. Specific questions posed by FDA are outlined beginning on the second page of the Federal Register notice: http://1.usa.gov/Y4ytgz The questions are also copied below as part of the draft agenda.
Submit written comments to: http://www.regulations.gov/#!documentDetail;D=FDA-2012-N-0962-0004 (Look for the “COMMENT NOW” button on the top right of the screen.).
Development of Safe and Effective Drug Therapies for Chronic Fatigue Syndrome (CFS)
and Myalgic Encephalomyelitis (ME)
April 25, 2013: Patient Focused Drug Development Workshop
1:00 p.m. Welcome
1:10 p.m. Background and goals of meeting
1:20 p.m. Overview of FDA’s Patient-Focused Drug Development initiative
1:30 p.m. Overview of discussion format
Panel 1: Disease Symptoms and Daily Impacts That Matter Most to Patients
Panel 1 Questions:
1. What are the most significant symptoms that you experience resulting from your condition? (Examples may include prolonged exhaustion, confusion, muscle pain, heat or cold intolerance, etc.)
2. What are the most negative impacts on your daily life that result from your condition and its symptoms? (Examples may include difficulty with specific activities, sleeping through the night, etc.)
- a. How does the condition affect your daily life on the best days and worst days?
- b. What changes have you had to make in your life because of your condition?
2:50 p.m. Break
Panel 2: Patient perspective on treating CFS and ME
Panel 2 Questions:
1. What treatments are you currently using to help treat your condition or its symptoms? (Consider prescription medicines, over-the-counter products and non-drug therapies such as activity limitations).
a. What specific symptoms do your treatments address?
b. How has your treatment regimen changed over time and why?
2. How well does your current treatment regimen treat the most significant symptoms of your disease?
- a. How well have these treatments improved your daily life (for example, improving your ability to do specific activities)?
- b. How well have these treatments worked for you as your condition has changed over time?
- c. What are the most significant downsides of these treatments (for example, specific side effects)?
4:15 p.m. Open public comment period
4:45 p.m. Closing remarks
5:00 p.m. Adjourn
April 26, 2013: Scientific Drug Development Workshop
Panel 1: Regulatory and Drug Development Innovation for CFS and ME
8:30 am Panel Presentations
Topics: background, drug innovation, repurposing drugs, regulatory pathways to expediting drug treatments
10:00 am Audience Question and Answer Period
10:20 am Break
Panel 2: Key Symptoms of CFS and ME (Patient and Clinician Panel)
10:30 am Panel Discussion
Topics: symptoms, outcome measures, disease heterogeneity
11:15 am Audience Question and Answer Period
11:30 am Lunch
Panel 3: CFS and ME Clinical Trial Endpoints and Design
12:30 pm Panel Presentations
Topics: clinical trial design, exercise challenge, outcome measures
2:30 pm Audience Question and Answer Period
3:00 pm Break
Panel 4: Summary and Scientific Gaps
3:15 pm Panel Discussion
Topics: key messages, scientific gaps, possible next steps
4:45 pm Audience Question and Answer Period
5:00 pm Closing Remarks
Confirmed Speakers, Panelists, and Moderators for Day 2 (April 26, 2013)
Lucinda Bateman, MD
Lisa Corbin, MD
Lily Chu, MD, MHSH (patient)
Jordan Dimitrakoff, MD, PhD
Nancy Klimas, MD
Dennis Mangan, PhD
Robert Miller (patient)
José Montoya, MD
Bernard Munos, PhD
Peter Rowe, MD
Christopher Snell, PhD
Jennie Spotila, JD (patient)
Elizabeth Unger, MD, PhD
Suzanne Vernon, PhD
Christine Williams, MEd (patient)
Note: Final agenda including titles of talks will be posted by the FDA approximately one week prior to the meeting. This agenda has been released by FDA for informational purposes only.
*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.March 21, 2013