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Reminder: Deadline to Submit Written Comments for Ataluren Ad Comm is Thursday, September 14

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.

Tips:

  • 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.

Learn more

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