On May 18, 2017, FDA’s Pediatric Advisory Committee will convene to consider the issue of allowing in-dwelling ports in Sarepta’s Essence trial. PPMD has been working closely with the FDA to plan this landmark meeting. Details, including date/time/location as well as instructions on submitting both written and oral testimony has now been published by the FDA in the Federal Register.
Information about the May 18 Pediatric Advisory Committee Meeting and how our Duchenne community can participate is included below.
The meeting will be held on May 18 from 8:30 AM – 5:30 PM in the Great Room of Building 31 at FDA in White Oak, Maryland.
The meeting will be webcast at https://collaboration.fda.gov/pacm051817/. There may be a limit on the number of people who can log on to the webcast, so we suggest logging on early to secure your spot.
8:30 AM – 5:30 PM (flexibility on ending time):
- Introductions of Committee
- FDA presentation of issue
- Presentations providing Issue Context & Background
- Presentation by a parent
- Open Public Hearing: 11 AM – 12:30 PM (90 minutes**)
- Sarepta presentation
- Presentation of Questions to the Committee
- Advisory Committee Discussion
Agenda & Meeting Materials
- Will the agenda and meeting materials be made available in advance of the meeting?
Yes, the Briefing documents will be posted on the FDA website in advance of the meeting and we will link to them as soon as they are available. The Briefing documents will include:
- The Referral from the IRB
- The Study Protocol
- Consents & Assents
- Background materials
- There is a 90 Minute Time Period Set Aside for Public Comment. Who Should Consider Making a Public Comment?
We encourage families who have had experience with ports through participation in the Sarepta Essence study to consider testifying. Please contact Ryan Fischer at email@example.com and Jordan McSherry at firstname.lastname@example.org for more information on how to register to speak at the advisory meeting and the availability of travel scholarships.
Written Public Comments
- Why Are Written Comments Important? How Are They Utilized?
Written comments are compiled and shared with the FDA Pediatric Advisory Committee members prior to the date of an Advisory Committee meeting. Thus, written comments are a critical part of helping to provide context to the Advisory Committee. It is important to note that the docket for this issue will remain open one day past the meeting. All comments will be taken into consideration before a final recommendation is forwarded to the FDA Commissioner.
- Who Should Consider Submitting Written Comments?
Any member of the public with experience and expertise in infusion therapies and/or in-dwelling ports in pediatrics, with special emphasis on Duchenne (Including Clinical experts)
- If someone is not able to attend the Advisory Committee meeting in person, is participation through the submission of written testimony a powerful method of participation?
- If someone is planning to present oral testimony during the Open Public Hearing on the day of the Advisory Committee Meeting, can they also submit written testimony?
- Can non US resident submit testimony?
- What types of information should be included in testimony?
The testimony you submit should reflect your direct experiences with Duchenne and the issue under review. Testimony from those who have been involved in the clinical trials as a participant, parent of a participant, clinician, researcher, or community member and have first-hand knowledge of how this issue impacts the daily life of individuals living with Duchenne are critical. Testimony from clinicians with expertise in this issue is also very important.
While the Pediatric Advisory Committee understands that – from an ethical perspective – parents are interested in protecting their children and make reasonable decisions, the testimony of families should include the first-hand considerations and experiences of parents and trials participants as you have weighed the decision to place a port.
Some considerations for families drafting testimony:
- Highlight that this is a decision that a parent wrestles with and ‘worries through’ – does not take lightly.
- It will be helpful for the Committee to hear how a parent approaches this decision and what considerations are made
- What are the benefits of a port – weighed against the risks of not having one?
- What are the trade-offs that you’ve had to consider?
Following the Meeting
- What happens following this meeting? Who does the Pediatric Ad Comm make a recommendation to? Is there a timeline for decision-making?
Following the meeting and the closing of the docket, a decisional memo will be sent from the Office of Pediatric Therapeutics to the FDA Commissioner. FDA Commissioner will then be briefed on the issue and will make a decision as to allow for an amendment to the protocol to allow for the option of an in-dwelling port – or not. The letter to the Commissioner will be public and posted on the FDA website, along with the Pediatric Advisory Committee meeting materials.
- If a recommendation is made to allow of an amended protocol – what happens next?
The decision is considered ‘permissive’. Each trial sites’ IRB would then have an opportunity to review the revised protocol and make a determination locally as to whether to amend their site’s protocol to allow for in-dwelling port use. A determination by the FDA would not automatically implement a change at every trial site.
Deadlines & Instructions
Written testimonies should be submitted on or before May 19th. The docket remains open one day following the Advisory Committee and all comments will be taken into full consideration -- but all comments received by May 5th will be forwarded to the Advisory Committee for their review PRIOR to the Advisory Committee meeting.
- How to Submit:
You will submit your testimony through an FDA portal, called a “docket”. Comments can be submitted starting Tuesday, April 25th. Click here to submit your comment. Please contact Ryan Fischer at email@example.com for assistance or questions about submitting written testimony.
Please raise your voice and join us.
Our community is hopeful.