What types of information should be included in written testimony?
Your direct experiences with Duchenne, and if applicable, with eteplirsen specifically. 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 eteplirsen 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 eteplirsen:
- Describe how Duchenne impacts you/your son, and your family
- What physical changes have you seen as a result of eteplirsen?
- 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 or stamina? Or changes in function? (i.e. teachers, family members, etc.)
- If at any point during the trial 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 he didn’t experience severe side effects, include that in your statement.
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 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 you/your son, you, and your family
- Describe what function you/your son wishes he could maintain and worries about losing over time
- Describe what meaningful benefit of a drug would be to you/your son and your family
Deadlines & Instructions:
Written testimony for the Sarepta Ad Comm must be submitted via email no later than January 7, 2016.
Send your email to: PCNS@fda.hhs.gov and cc MakeDuchenneHistory@gmail.com
Email subject line: Written testimony submission for FDA Advisory Committee Meeting to consider Eteplirsen – January 22, 2016
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 – January 22, 2016.”
If you do not have direct experience with Duchenne or eteplirsen, but are interested in supporting our effort, we do need you! Please consider attending the January 22nd FDA Advisory Committee Meeting in White Oak. Details about attending the meeting and traveling to White Oak can be found here .
Please help us ensure that the strength of our Duchenne community is once again felt by federal policymakers on January 22nd.