On September 2nd, PPMD convened a public webinar for the Duchenne community focused on FDA Advisory Committee Meetings (or “Ad Comms”). After working closely with representatives from FDA, we are excited to share some of the frequently asked questions that arise regarding the Ad Comm process, as well as some specifically relevant to our community. As we get closer to possible dates for these meetings, we will add information as it becomes available.

Please note: although we anticipate that our community will have two Ad Comms in the end of November, no dates have been confirmed on the FDA calendar so we are in a bit of a holding pattern until the agency notifies the companies. Until then, PPMD will continue to work with our partners at both BioMarin and Sarepta to help them prepare for these critical meetings. In the meantime, make sure you check out the FAQs below. We also encourage you to watch the recording at your convenience if you were unable to attend.

What is the FDA Advisory Committee?

• The Peripheral and Central Nervous System Drugs Advisory Committee reviews and evaluates data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of neurologic diseases and makes appropriate recommendations to the Commissioner of Food and Drugs.
• Part of the FDA mission is to evaluate new therapies and determine which are safe and effective for their intended use.
• The Committee is advisory in nature, providing recommendations to the agency; FDA makes the final decisions.
• The Peripheral and Central Nervous System Drugs Advisory Committee is one of 48 technical and scientific advisory committees and panels; it would be responsible for reviewing potential treatments for Duchenne are considered by.

How is an Advisory Committee Meeting announced?

• Meetings are officially announced through the Federal Register at least 15 days in advance of the meeting.
• The announcement will also be posted on the FDA Patient Network FDA.gov/ForPatients (Calendar of Public Meetings).
• Announcement includes date(s), times, location, topic of discussion, web links for further information, and instructions & contacts for written or in-person comments from the public.

What occurs on the actual Advisory Committee Meeting?

The entire proceeding is open to the public and – when held on the FDA campus – are streamed live on the web.  Web access information is made available through the Federal Register Notice, and through the Calendar of Public Meetings. While the public (and the Duchenne community) are encouraged to attend the full meeting, active participation by the public in the meeting is only permitted during the designated Open Public Hearing.

The typical Ad Comm schedule:

Members of the Public and Duchenne community are encouraged to participate in 3 critical ways:

1) Submit Written Testimony

• Written comments are a powerful and important way to participate in the Ad Comm process, regardless of whether or not you are able to physically be present on the day of the meeting.
• To be considered, all written comments should be submitted to the designated Agency official in meeting announcement by the deadline date in the Federal Register Notice, either by mail, FAX, or e-mail.

2) Submit Oral Testimony during the Open Public Hearing *

• To speak during the open public hearing, interested persons need to contact FDA by deadline in the Federal Register announcement with:
  • a brief statement of the general nature of the evidence or arguments you wish to present
  • the names and addresses of proposed participants, and
  • the approximate amount of time requested to make your presentation
• A confirmation will be sent to you indicating that your testimony will be among those included during the Open Public Hearing.
• When you arrive at the meeting on the day of the Ad Comm, speakers are asked to identify themselves to FDA staff members and provide the staff with any handouts or other materials for distribution to committee members, and be included in the meeting’s permanent record.
• Designated speakers will be assigned a number to establish order of speakers.
• Special seating is reserved for registered speakers from the public.
• Those who speak are encouraged to disclose any financial relationships they may have with the topic of the meeting or with the sponsors or competitors of the products under discussion.
• At least one hour is set aside for the Open Public Hearing.
• FDA asks people with similar messages to consolidate and present together, if possible.
• In rare instances where public demand is high, FDA may opt to implement a lottery and randomly select from among the requests to speak.
• A table will be made available near the registration desk for any printed materials you wish to make available to those attending the meeting.
• For speakers who’ve been accepted to present oral testimony at the Open Public Hearing who would like to include video during your testimony:
  • should send it on CD (DVD-ROM or CD-ROM) in MPEG-4 format (.mp4, .m4v, and other Microsoft MPEG-4) or Windows Media Player Format (.asf, .wma, .wmv, .wm)
  • all videos should be sent at least a week prior to the Ad Comm where you will be participating in the Open Public Hearing so that playability can be ensured, and corrected if there are any technical issues
  • Due to software security concerns, videos will not be accepted it on a thumb drive the day of the Ad Comm

3) Comprise Audience (day of Ad Comm meeting)

• This is a historic moment for our Duchenne community and we encourage all those interested in attending the Ad Comm meeting to attend in-person.
• Only those who have submitted their intention to present oral testimony - and have received confirmation of acceptance – will be able to speak on the day of the Ad Comm during the designated Open Public Hearing.
• Audience members may not present comments or questions to the Committee unless recognized by the Chair.
• Attendees will be subject to security screening on entering, including passing through metal detectors, and x-ray inspection of briefcases, packages, etc.
• In order to ensure that we can accommodate all participants, please notify PPMD or FDA personnel (listed as contact within Federal Register), at least 7 days in advance, of any physical accommodations you may need due to disability. PPMD staff will work to coordinate with FDA personnel.
• FDA is committed to the orderly conduct of its advisory committee meetings. Please visit the FDA Web site at Public Conduct During FDA Advisory Committee Meetings for procedures on public conduct during advisory committee meetings.
• Directions for public meetings at the FDA White Oak Campus.
  
  

Staying engaged:

• PPMD will be continuing to disseminate information about the Ad Comms as it becomes available.
• Background materials related to the meeting will be posted on the FDA web site prior to the meeting:
  • Meeting agenda
  • Instructions for providing your comments
  • Roster of committee participants
  • Briefing information submitted by the sponsor and FDA
  • Presentation slides
  • Questions for the committee
  • Webcast information

Resources:

• PPMD/FDA Ad Comm Webinar Recording (September 2, 2015)
• PPMD/FDA Ad Comm Webinar slide deck [PDF download]
• FDA Patient Network
  
• Ad Comm Webcast link