The deadline to submit a written comment for the FDA Advisory Committee Meeting on ataluren is this upcoming Thursday, September 14th.
To make a comment, view the notice document and click COMMENT NOW.
Below are some tips and things to think about when drafting remarks.
- Brevity—advisors are given a lot of information. Letters are among the last information they read.
- A clear POV up front—again, advisors get a lot of information so a position statement embedded in a paragraph on the third page of a letter may not get noticed.
- Focus on a few main messages.
- Bullets and/or bolding the keys points you want to make (it also helps make the letter “skimmable”).
- Consider asking a family member, teacher, friend, or health professional who has seen from their point of view how the drug has impacted you/your son.
Things to think about for drafting your submission:
For those with experience on the product:
- Describe how Duchenne impacts you/your child and your family.
- Mention what study you (or your son) was in and how long on drug.
- What physical changes have you seen as a result of the experimental product?
- If you/our son was in an early study and stopped then started the drug again, be sure to mention this as well and whether you saw changes when stopped.
- 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.
For those outside of the trial/experience with the 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?
- Describe how Duchenne impacts you, your child, 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 child and your family.