FDA’s Draft Agenda for the CFS/ME Patient Input Meeting
This article was originally published in RPM Report
Executive Summary
Here is a draft agenda made available by FDA ahead of the April 25-26 first-ever PDUFA V patient input workshop on chronic fatigue syndrome/myalgic encephalomyelitis. FDA notes that a final agenda will be made available a week before the meeting.
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:
- 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.)
- 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.)
- How does the condition affect your daily life on the best days and worst days?
- 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:
- 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).
- What specific symptoms do your treatments address?
- How has your treatment regimen changed over time and why?
- How well does your current treatment regimen treat the most significant symptoms of your disease?
- How well have these treatments improved your daily life (for example, improving your ability to do specific activities)?
- How well have these treatments worked for you as your condition has changed over time?
- 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
Scientific Drug Development Workshop Confirmed Speakers, Panelists, and Moderators
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)