Why Are Written Comments Important? How Are They Utilized?
Written comments are compiled and shared with the FDA Advisory Committee members prior to the date of a product’s/ company’s Advisory Committee meeting. Thus, written comments are a critical part of helping to provide context to the Advisory Committee.
Who Should Consider Submitting Written Comments?
Any member of the public with a vested interest in the product’s review, and /or experience and expertise in the disorder that the therapeutic product has been designed to benefit.
- 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?Yes!
- If someone is able to attend the Advisory Committee meeting in person but is not planning to make a public statement at the Open Public Hearing on the day of the Advisory Committee, is participation through the submission of written testimony a powerful method of participation?Yes!
- 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?Yes!
- Can non US resident submit testimony?
What types of information should be included in written testimony?
The written testimony you submit should reflect your direct experiences with Duchenne and the product under review. The Advisory Committee will be presented with a vast amount of data about the experimental product and is being asked to determine whether enough safety and efficacy data has been made available to warrant access to the public. 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 product has impacted the daily life of individuals living with Duchenne are critical. Testimony from community members who are awaiting access to these therapies is also very important.
Points for consideration:
For those with experience on the product:
- What physical changes have you seen as a result of the experimental product?
- What activities of daily living did you/your child have difficulty performing prior to the trial’s initiation that became easier or possible as the trial went on?
- Are there people in you/your child’s daily life who may have been unaware of the trial and made observations that spoke to increased endurance and stamina or function? (i.e. teachers, family members, etc)
- If at any point during the trial you/ your child experienced side effects related to the experimental product, describe those – and discuss whether you considered them to be severe and how that played into your decision to continue/discontinue participation in the trial. If you have not seen any side effects please indicate that.
- Describe how Duchenne impacts you/your child and your family
For those outside of the trial/experience with product some things to think about for your submission:
- As the Advisory Committee members prepare to listen to drug sponsors and the public, what do you want them to most know about your child and others in the Duchenne community?
- If your child was not included in the current trial, but is eligible for this therapy if approved – what would you like for the FDA Advisory Committee to know prior to this review?
- If you/your child is not eligible for this particular therapy, but would be eligible for a therapy that could soon be in development or is in early testing – what would you like the FDA Advisory Committee to know prior to this review?
Describe how Duchenne impacts your son, you, and your family
Describe what function your son wishes he could maintain and worries about losing over time
Describe what meaningful benefit of a drug would be to your son and your family
Deadlines & Instructions
For the Eteplirsen AdComm
Written testimonies should be submitted on or before April 11th
Send your email to: PCNS@fda.hhs.gov
Email subject line: Written testimony submission for FDA Advisory Committee Meeting to consider Eteplirsen
Attn: Moon Hee V. Choi
Please be sure to indicate “The following is my written testimony submission for the FDA Advisory Committee Meeting to consider Eteplirsen – April 25, 2016”
Please let us know if we can help in any way. Please consider cc’ing email@example.com, we’d like to compile all them.